1 00:00:05,840 --> 00:00:06,760 >>I CAN'T TELL WHAT YOU A GREAT 2 00:00:06,760 --> 00:00:10,800 PLEASURE IT IS TO SEE EVERYONE 3 00:00:10,800 --> 00:00:11,000 HERE. 4 00:00:11,000 --> 00:00:13,880 NON-PROFIT FORUM IS REALLY A 5 00:00:13,880 --> 00:00:16,400 HIGHLIGHT FOR NINDS, CERTAINLY, 6 00:00:16,400 --> 00:00:17,360 EVERY YEAR. 7 00:00:17,360 --> 00:00:20,000 AND IT'S GREAT TO SEE PEOPLE 8 00:00:20,000 --> 00:00:23,920 OUTSIDE OF THE SQUARES AND IN 9 00:00:23,920 --> 00:00:24,920 PERSON. 10 00:00:24,920 --> 00:00:27,280 I THINK A LOT OF THE 11 00:00:27,280 --> 00:00:28,640 INTERACTIONS WE'VE HAD OVER THE 12 00:00:28,640 --> 00:00:30,160 YEARS HAVE BEEN QUITE VALUABLE 13 00:00:30,160 --> 00:00:34,000 AND I'M HOPING THAT THIS KIND OF 14 00:00:34,000 --> 00:00:37,080 HYBRID BOTH ALLOWS LOTS OF 15 00:00:37,080 --> 00:00:39,360 PEOPLE TO BE INVOLVED AND PEOPLE 16 00:00:39,360 --> 00:00:41,000 WHO TRAVEL IN HOPEFULLY CAN GET 17 00:00:41,000 --> 00:00:47,480 INTO SOME REALLY GOOD 18 00:00:47,480 --> 00:00:49,520 CONVERSATIONS THAT MOVE YOUR 19 00:00:49,520 --> 00:00:52,200 AGENDAS FORWARD AND OUR COMMON 20 00:00:52,200 --> 00:00:54,640 AGENDA WHICH IS TO FIND MORE 21 00:00:54,640 --> 00:00:56,360 EFFECTIVE TREATMENTS FOR A 22 00:00:56,360 --> 00:00:58,960 VARIETY OF NEUROLOGIC DISEASES. 23 00:00:58,960 --> 00:01:00,400 UNFORTUNATELY, WE SHOULD ALWAYS 24 00:01:00,400 --> 00:01:02,560 START BY APOLOGIZING FOR LACK OF 25 00:01:02,560 --> 00:01:04,920 EFFECTIVE THERAPIES FOR MANY OF 26 00:01:04,920 --> 00:01:06,120 THE DISEASES. 27 00:01:06,120 --> 00:01:08,880 BUT OUR INSTITUTE AND PEOPLE WHO 28 00:01:08,880 --> 00:01:10,600 WORK HERE AND YOU ARE ALL 29 00:01:10,600 --> 00:01:13,240 DEDICATED TO TRY AND CHANGE THAT 30 00:01:13,240 --> 00:01:16,920 AND I THINK WE'LL HEAR TODAY 31 00:01:16,920 --> 00:01:19,440 THAT THERE ARE THESE WINDOWS 32 00:01:19,440 --> 00:01:22,640 OPENING UP FOR NEW THERAPIES, SO 33 00:01:22,640 --> 00:01:23,920 YOU'LL HERE THAT PARTICULARLY IN 34 00:01:23,920 --> 00:01:29,440 THE DISCUSSION TODAY ABOUT GENE 35 00:01:29,440 --> 00:01:29,920 THERAPIES. 36 00:01:29,920 --> 00:01:32,160 THESE ARE REALLY KIND OF CUTTING 37 00:01:32,160 --> 00:01:32,600 EDGE. 38 00:01:32,600 --> 00:01:35,040 IT'S A REALLY NEW AREA OF 39 00:01:35,040 --> 00:01:37,560 SCIENCE, TO TELL YOU THE TRUTH, 40 00:01:37,560 --> 00:01:40,040 THAT WE HAVE GREAT EXPECTATIONS 41 00:01:40,040 --> 00:01:40,200 FOR. 42 00:01:40,200 --> 00:01:41,960 BUT IT'S GOING TO BE A PROCESS 43 00:01:41,960 --> 00:01:45,080 OF LEARNING HOW TO DO THIS, BOTH 44 00:01:45,080 --> 00:01:49,440 REPLACING GENES THAT ARE MISSING 45 00:01:49,440 --> 00:01:52,680 IN PEOPLE WHO HAVE NEUROGENETIC 46 00:01:52,680 --> 00:01:53,960 DISORDERS AND TURNING OFF GENES 47 00:01:53,960 --> 00:01:55,840 THAT ARE MUTATED AND OF COURSE 48 00:01:55,840 --> 00:01:57,040 TROUBLE, BUT BOTH TECHNOLOGIES 49 00:01:57,040 --> 00:01:57,320 NOW EXIST. 50 00:01:57,320 --> 00:02:01,200 AND SO ON THE ONE HAND IT'S AN 51 00:02:01,200 --> 00:02:02,840 ENGINEERING PROBLEM OF MAKING IT 52 00:02:02,840 --> 00:02:04,760 HAPPEN IN AN EFFECTIVE WAY, BUT 53 00:02:04,760 --> 00:02:06,560 ALSO LEARNING A LOT ABOUT THE 54 00:02:06,560 --> 00:02:08,880 SAFETY ISSUES AS WE MOVE 55 00:02:08,880 --> 00:02:09,120 FORWARD. 56 00:02:09,120 --> 00:02:12,440 A LOT OF THE DRUGS THAT WE'VE 57 00:02:12,440 --> 00:02:14,560 BEEN TRYING, SOMETIMES THEY HAVE 58 00:02:14,560 --> 00:02:16,320 SOME EFFECT, SOMETIMES THEY 59 00:02:16,320 --> 00:02:17,760 DON'T HAVE ANY EFFECT. 60 00:02:17,760 --> 00:02:19,760 BUT THEY DON'T OFTEN HOLD THE 61 00:02:19,760 --> 00:02:23,320 PROMISE OF REALLY, YOU KNOW, A 62 00:02:23,320 --> 00:02:25,000 CURE AS NEW GENE THERAPIES DO. 63 00:02:25,000 --> 00:02:30,360 SO WE'RE GOING TO HEAR TODAY 64 00:02:30,360 --> 00:02:31,240 FROM TERRY PIROVOLAKIS, A 65 00:02:31,240 --> 00:02:36,400 CHAMPION IN MOVING THIS FIELD 66 00:02:36,400 --> 00:02:37,800 FORWARD, AND SUCCESS STORIES. 67 00:02:37,800 --> 00:02:40,640 WE DID HAVE IN OUR DISORDERS WE 68 00:02:40,640 --> 00:02:42,160 HAD THREE DIFFERENT NEW DRUGS 69 00:02:42,160 --> 00:02:44,560 APPROVED OVER THE LAST YEAR, 70 00:02:44,560 --> 00:02:45,800 NEUROLOGIC DISORDERS, SO YOU'LL 71 00:02:45,800 --> 00:02:49,600 BE HEARING SUCCESS STORIES 72 00:02:49,600 --> 00:02:49,800 THERE. 73 00:02:49,800 --> 00:02:52,480 AND THEN I THINK WE'RE HOPING 74 00:02:52,480 --> 00:02:58,160 THAT AS ALWAYS, THIS IS A WAY IN 75 00:02:58,160 --> 00:03:00,440 WHICH THE NON-PROFIT 76 00:03:00,440 --> 00:03:01,200 ORGANIZATIONS CAN COMMUNICATE 77 00:03:01,200 --> 00:03:01,880 WITH NINDS. 78 00:03:01,880 --> 00:03:03,160 NINDS CAN TELL YOU KIND OF WHAT 79 00:03:03,160 --> 00:03:04,720 WE'RE UP TO. 80 00:03:04,720 --> 00:03:07,480 BUT WE REALLY WANT TO LISTEN TO 81 00:03:07,480 --> 00:03:08,120 YOU. 82 00:03:08,120 --> 00:03:10,400 YOU ARE ACTUALLY OUR 83 00:03:10,400 --> 00:03:10,840 CONSTITUENCY. 84 00:03:10,840 --> 00:03:12,480 SO AS YOU KNOW, NINDS, WE'RE 85 00:03:12,480 --> 00:03:15,640 VERY HAPPY TO TAKE YOUR 86 00:03:15,640 --> 00:03:17,960 DONATIONS EVERY APRIL 15. 87 00:03:17,960 --> 00:03:19,400 SO KEEP PAYING YOUR TAXES. 88 00:03:19,400 --> 00:03:23,440 BUT THE IDEA IS IT'S NOT OUR 89 00:03:23,440 --> 00:03:24,080 MONEY. 90 00:03:24,080 --> 00:03:25,280 IT'S THE TAXPAYERS' MONEY, AND 91 00:03:25,280 --> 00:03:27,040 THE PURPOSE IS TO MOVE THE 92 00:03:27,040 --> 00:03:28,560 THERAPIES FORWARD FOR YOUR 93 00:03:28,560 --> 00:03:28,840 DISEASES. 94 00:03:28,840 --> 00:03:30,440 SO WE REALLY WANT TO HEAR FROM 95 00:03:30,440 --> 00:03:33,480 YOU, AS WELL AS TELL YOU WHAT 96 00:03:33,480 --> 00:03:36,760 OUR THOUGHTS ARE GOING FORWARD. 97 00:03:36,760 --> 00:03:41,720 AND OF COURSE IT'S A REAL 98 00:03:41,720 --> 00:03:45,120 PARTNERSHIP AND THIS IS REALLY 99 00:03:45,120 --> 00:03:47,360 THE MEETING IN WHICH WE KIND OF 100 00:03:47,360 --> 00:03:49,560 GET TO HEAR FROM SO MANY 101 00:03:49,560 --> 00:03:51,880 DIFFERENT GROUPS. 102 00:03:51,880 --> 00:03:53,280 AND THEN ALSO A CHANCE FOR 103 00:03:53,280 --> 00:03:55,240 DIFFERENT GROUPS TO TALK TO EACH 104 00:03:55,240 --> 00:03:57,120 OTHER AND GET LESSONS LEARNED. 105 00:03:57,120 --> 00:03:58,440 EACH OF HAVE YOU YOUR OWN 106 00:03:58,440 --> 00:04:01,280 CHALLENGES AS YOU MOVE YOUR 107 00:04:01,280 --> 00:04:04,040 DISEASE AREA FORWARD AND YOUR 108 00:04:04,040 --> 00:04:05,680 FOUNDATIONS FORWARD, AND THERE'S 109 00:04:05,680 --> 00:04:08,200 A MIX OF PEOPLE HERE DOING IT 110 00:04:08,200 --> 00:04:09,200 FOR MANY DECADES, SOME PEOPLE 111 00:04:09,200 --> 00:04:09,720 STARTING UP. 112 00:04:09,720 --> 00:04:11,920 SO IT'S ALSO A CHANCE TO LISTEN 113 00:04:11,920 --> 00:04:13,440 TO EACH OTHER. 114 00:04:13,440 --> 00:04:14,960 EVERYBODY HERE IS INCREDIBLY 115 00:04:14,960 --> 00:04:16,720 GENEROUS WITH THEIR THOUGHTS AND 116 00:04:16,720 --> 00:04:18,960 TIME, I'M SURE WOULD LOVE TO 117 00:04:18,960 --> 00:04:25,600 HELP YOU AS YOU MOVE THROUGH 118 00:04:25,600 --> 00:04:27,200 YOUR FOUNDATION PROGRESS. 119 00:04:27,200 --> 00:04:29,000 SO WITH THAT I'D LIKE TO TURN IT 120 00:04:29,000 --> 00:04:36,000 OAF TO OUR KEYNOTE SPEAKER, 121 00:04:36,000 --> 00:04:37,080 TERRY PIROVOLAKIS, TO TELL THE 122 00:04:37,080 --> 00:04:39,280 STORY OF WHAT GOT HIM INTO THIS 123 00:04:39,280 --> 00:04:41,880 IN THE FIRST PLACE, WHICH IS A 124 00:04:41,880 --> 00:04:45,200 VERY POIGNANT STORY. 125 00:04:45,200 --> 00:04:48,040 BUT I THINK THE OTHER PART IS 126 00:04:48,040 --> 00:04:50,120 HOW TERRY WAS ABLE TO SEE FROM 127 00:04:50,120 --> 00:04:51,760 HOW HE GOT INTO THIS IN THE 128 00:04:51,760 --> 00:04:53,840 BEGINNING HOW IT COULD BE 129 00:04:53,840 --> 00:04:55,760 SOMETHING MUCH, MUCH BIGGER. 130 00:04:55,760 --> 00:04:57,360 AND WE'VE BEEN REALLY OVERJOYED 131 00:04:57,360 --> 00:05:00,040 TO BE ABLE TO WORK WITH HIM AND 132 00:05:00,040 --> 00:05:01,360 LOOK FORWARD TO WORKING WITH HIM 133 00:05:01,360 --> 00:05:05,520 IN THE FUTURE ON PUSHING A 134 00:05:05,520 --> 00:05:07,920 NUMBER OF GENE THERAPIES INTO 135 00:05:07,920 --> 00:05:10,040 CLINICAL TRIALS. 136 00:05:10,040 --> 00:05:15,920 SO, TERRY? 137 00:05:15,920 --> 00:05:16,960 >> HI, EVERYBODY. 138 00:05:16,960 --> 00:05:19,400 IT'S A REAL HONOR TO BE HERE. 139 00:05:19,400 --> 00:05:21,520 FOUR YEARS AGO, I WAS IN A ROOM 140 00:05:21,520 --> 00:05:24,480 JUST LIKE THIS ONE DOWNSTAIRS, 141 00:05:24,480 --> 00:05:28,120 JUST ONE MONTH GREEN INTO THIS 142 00:05:28,120 --> 00:05:29,200 NEW EXPERIENCE I WAS GOING 143 00:05:29,200 --> 00:05:30,840 THROUGH TO SAVE MY SON MICHAEL. 144 00:05:30,840 --> 00:05:33,240 IT'S A TRUE HONOR TO BE HERE 145 00:05:33,240 --> 00:05:34,560 TALKING ABOUT MY JOURNEY AND 146 00:05:34,560 --> 00:05:38,120 SUPPORT FROM NIH AND EVERYBODY 147 00:05:38,120 --> 00:05:39,280 THAT SUPPORTED ME THROUGH THE 148 00:05:39,280 --> 00:05:41,880 CRAZY DREAM I'M GOING TO TALK TO 149 00:05:41,880 --> 00:05:42,560 YOU ABOUT. 150 00:05:42,560 --> 00:05:43,520 IT'S A GREAT HONOR. 151 00:05:43,520 --> 00:05:44,000 HANG YOU -- THANK YOU FOR HAVING ME 152 00:05:44,000 --> 00:05:44,960 I'M TERRY PIROVOLAKIS. 153 00:05:44,960 --> 00:05:48,000 THE LAST 20 YEARS OF MY LIFE 154 00:05:48,000 --> 00:05:50,320 I'VE BEEN AN I.T. DIRECTOR FOR 155 00:05:50,320 --> 00:05:51,440 LARGE BANKS ACROSS CANADA. 156 00:05:51,440 --> 00:05:57,440 I'LL TELL A STORY ABOUT MY SON'S 157 00:05:57,440 --> 00:05:58,600 JOURNEY, MY FAMILY'S JOURNEY, 158 00:05:58,600 --> 00:06:00,280 FOR A CURE FOR MY SON. 159 00:06:00,280 --> 00:06:03,560 MY WIFE IS GEORGIA, MY DAUGHTER 160 00:06:03,560 --> 00:06:07,160 ZOE, MIDDLE SON ZACH, THE GRUMPY 161 00:06:07,160 --> 00:06:10,240 FUR BALL IS MICHAEL. 162 00:06:10,240 --> 00:06:16,160 HE WAS BORN DECEMBER 17, 2017. 163 00:06:16,160 --> 00:06:18,440 A NORMAL HEALTHY BIRTH, HOME IN 164 00:06:18,440 --> 00:06:19,400 THREE HOURS. 165 00:06:19,400 --> 00:06:21,280 WE THOUGHT THE LIFE WOULD 166 00:06:21,280 --> 00:06:25,880 PROGRESS LIKE THE OTHER 167 00:06:25,880 --> 00:06:26,240 CHILDREN. 168 00:06:26,240 --> 00:06:29,960 FIVE MONTHS AFTER BIRTH WASN'T 169 00:06:29,960 --> 00:06:31,280 LIFTING HIS HEAD, 10th 170 00:06:31,280 --> 00:06:32,280 PERCENTILE FOR HEAD GROWTH. 171 00:06:32,280 --> 00:06:35,760 KEEP AN EYE ON HIM BUT SHOULD 172 00:06:35,760 --> 00:06:36,200 OUTGROW IT. 173 00:06:36,200 --> 00:06:37,280 DON'T BE CONCERNED. 174 00:06:37,280 --> 00:06:41,240 AT SIX MONTHS AFTER BIRTH, WE 175 00:06:41,240 --> 00:06:43,400 VISITED A PEDIATRICIAN, WE 176 00:06:43,400 --> 00:06:44,640 STARTED DOING INTENSE PHYSICAL 177 00:06:44,640 --> 00:06:46,360 THERAPY, AND NOTICED HE HAD 178 00:06:46,360 --> 00:06:47,560 LITTLE OF TONE. 179 00:06:47,560 --> 00:06:49,240 HIS HEAD WAS IN THE FIFTH 180 00:06:49,240 --> 00:06:53,400 PERCENTILE AT THAT POINT IN 181 00:06:53,400 --> 00:06:53,720 TIME. 182 00:06:53,720 --> 00:06:57,240 HE STARTED REACHING FOR THINGS, 183 00:06:57,240 --> 00:06:58,520 NO BIG DEAL. 184 00:06:58,520 --> 00:07:00,440 AND WE CONTINUED ON AND THOUGHT 185 00:07:00,440 --> 00:07:06,440 SOMETHING THAT HE WILL OUTGROW. 186 00:07:06,440 --> 00:07:07,520 EIGHT MONTHS AFTER BIRTH, 187 00:07:07,520 --> 00:07:09,480 INFECTIOUS DISEASE, I WAS DOING 188 00:07:09,480 --> 00:07:10,800 WORK IN LATIN AMERICA, THOUGHT 189 00:07:10,800 --> 00:07:11,800 MAYBE HE CONTRACTED ZIKA. 190 00:07:11,800 --> 00:07:13,640 WE DID ALL THE TEST THERE IS. 191 00:07:13,640 --> 00:07:16,320 AT THIS POINT HE STILL WASN'T 192 00:07:16,320 --> 00:07:18,240 REACHING MILESTONES, WELL 193 00:07:18,240 --> 00:07:18,480 BEHIND. 194 00:07:18,480 --> 00:07:22,840 HIS HEAD WAS AT THE 3rd 195 00:07:22,840 --> 00:07:25,400 PERCENTILE BUT SITTING UP AND 196 00:07:25,400 --> 00:07:27,120 CONTINUING TO MOVE ALONG. 197 00:07:27,120 --> 00:07:30,440 AT TEN MONTHS ALL THE INFECTIOUS 198 00:07:30,440 --> 00:07:33,640 DISEASES CAME BACK RULED OUT. 199 00:07:33,640 --> 00:07:37,920 NEUROLOGY DID AN MRI SEVERE 200 00:07:37,920 --> 00:07:41,760 MICROCEPHALY, ABNORMAL WHITE 201 00:07:41,760 --> 00:07:43,880 MATTER, TONE WAS IMPROVING. 202 00:07:43,880 --> 00:07:46,280 WE KNEW SOMETHING WAS WRONG AND 203 00:07:46,280 --> 00:07:47,920 PRAYED MICHAEL WOULD GET BETTER 204 00:07:47,920 --> 00:07:49,760 BUT THIS WAS SOMETHING WE 205 00:07:49,760 --> 00:07:51,520 COULDN'T PRAY AWAY. 206 00:07:51,520 --> 00:07:54,040 AT 12 MONTHS WE RULED OUT ALL 207 00:07:54,040 --> 00:07:58,320 THE DISEASES ON THE PANELS. 208 00:07:58,320 --> 00:08:00,520 HE WAS NEGATIVE 3 PERCENTILE IN 209 00:08:00,520 --> 00:08:01,200 HEAD GROWTH. 210 00:08:01,200 --> 00:08:04,120 HE BEGAN PULL TO STAND, BEGAN TO 211 00:08:04,120 --> 00:08:05,320 CRAWL. 212 00:08:05,320 --> 00:08:06,440 HE WAS MAKING IMPROVEMENTS. 213 00:08:06,440 --> 00:08:09,160 WE WERE SENT TO NEUROLOGY AT 214 00:08:09,160 --> 00:08:13,080 THIS POINT TO DO A REPORT. 215 00:08:13,080 --> 00:08:16,720 ON APRIL 2, 2019, MICHAEL 216 00:08:16,720 --> 00:08:19,000 DIAGNOSED WITH SPASTIC 217 00:08:19,000 --> 00:08:20,960 PARAPLEGIA TYPE 50, TOLD THERE 218 00:08:20,960 --> 00:08:30,680 WAS VERY LITTLE KNOWN, IT CAUSES 219 00:08:30,680 --> 00:08:35,000 MICROCEPHALY AND EPILEPSY, 220 00:08:35,000 --> 00:08:36,280 QUADRIPLEGIA BY SECOND DECADE. 221 00:08:36,280 --> 00:08:40,120 I WAS IN A FOG, WHAT'S GOING ON. 222 00:08:40,120 --> 00:08:41,440 GET THE EMERGENCY ROOM READY, 223 00:08:41,440 --> 00:08:43,960 WE'LL TAKE WHATEVER WE NEED OUT 224 00:08:43,960 --> 00:08:44,160 OF ME. 225 00:08:44,160 --> 00:08:48,800 THE DOCTOR SAID SOMETHING YOU 226 00:08:48,800 --> 00:08:50,880 CAN'T DO WITH A GENETIC 227 00:08:50,880 --> 00:08:53,520 CONDITION, GO HOME AND LOVE HIM, 228 00:08:53,520 --> 00:08:54,600 ENJOY HIS LIFE AND THROUGH 229 00:08:54,600 --> 00:08:59,320 THERAPY TRY TO MOVE HIM ALONG. 230 00:08:59,320 --> 00:09:01,160 WE WERE TOLD SAVING MICHAEL WAS 231 00:09:01,160 --> 00:09:01,760 IMPOSSIBLE. 232 00:09:01,760 --> 00:09:04,320 FOR US IMPOSSIBLE WAS DOING 233 00:09:04,320 --> 00:09:04,560 NOTHING. 234 00:09:04,560 --> 00:09:10,000 WE KNEW WE WOULD GO ANYWHERE, DO 235 00:09:10,000 --> 00:09:16,920 ANYTHING, GIVE EVERYTHING TO 236 00:09:16,920 --> 00:09:18,040 SAVE MICHAEL. 237 00:09:18,040 --> 00:09:19,560 I DECIDED I THERE WAS NOTHING 238 00:09:19,560 --> 00:09:21,000 GOING TO STOP ME TO FIND A 239 00:09:21,000 --> 00:09:22,080 TREATMENT FOR MY SON. 240 00:09:22,080 --> 00:09:24,600 I CAME TO THE NIH. 241 00:09:24,600 --> 00:09:27,800 YOU CAN SEE THAT'S FOUR YEARS 242 00:09:27,800 --> 00:09:31,320 ALMOST TO THE DAY, I WENT TO 243 00:09:31,320 --> 00:09:33,720 SHEFFIELD, MET AN EXPERT IN GENE 244 00:09:33,720 --> 00:09:35,800 THERAPY, PUT UP A POSTER, 245 00:09:35,800 --> 00:09:38,400 WANTED, A CURE FOR MICHAEL. 246 00:09:38,400 --> 00:09:40,520 I MET SIX OF THE SEVEN WORLD 247 00:09:40,520 --> 00:09:42,440 EXPERTS, PEOPLE AT NIH, FDA. 248 00:09:42,440 --> 00:09:44,040 I ASKED IF YOU WERE TO TREAT 249 00:09:44,040 --> 00:09:46,000 YOUR SON OR YOUR CHILD, WHAT 250 00:09:46,000 --> 00:09:47,440 WOULD YOU DO? 251 00:09:47,440 --> 00:09:48,760 THEY ALL SAID GENE THERAPY. 252 00:09:48,760 --> 00:09:50,280 THE SECOND THING I SAID, WHO 253 00:09:50,280 --> 00:09:52,800 WOULD YOU GO WITH? 254 00:09:52,800 --> 00:09:53,680 ONE RECOMMENDATION, WE'LL GET TO 255 00:09:53,680 --> 00:09:55,760 IN A MINUTE. 256 00:09:55,760 --> 00:09:59,520 I ATTENDED FOOD CAMP AND EVERY 257 00:09:59,520 --> 00:10:04,320 CONFERENCE FOR THE NEXT TWO 258 00:10:04,320 --> 00:10:06,280 YEARS. 259 00:10:06,280 --> 00:10:08,120 WE COLLABORATED WITH NIH. 260 00:10:08,120 --> 00:10:09,000 REVERSE MORTGAGED OUR HOME, 261 00:10:09,000 --> 00:10:11,400 SPENT OUR LIFE SAVINGS TO GET 262 00:10:11,400 --> 00:10:12,040 THINGS STARTED. 263 00:10:12,040 --> 00:10:13,720 A MONTH AND A HALF TO THE DAY 264 00:10:13,720 --> 00:10:15,360 THAT MICHAEL WAS DIAGNOSED, WE 265 00:10:15,360 --> 00:10:17,440 HIRED DR. GRAY AND DR. CHEN AT 266 00:10:17,440 --> 00:10:19,480 THE UNIVERSITY OF TEXAS 267 00:10:19,480 --> 00:10:20,840 SOUTHWESTERN MEDICAL CENTER TO 268 00:10:20,840 --> 00:10:22,880 MAKE US THE GENE THERAPY WE WERE 269 00:10:22,880 --> 00:10:23,760 LOOKING FOR. 270 00:10:23,760 --> 00:10:25,200 AT THIS POINT UNFORTUNATELY WE 271 00:10:25,200 --> 00:10:28,960 REALIZED WE HAD TO RAISE $3 272 00:10:28,960 --> 00:10:33,520 MILLION, AT LEAST $3 MILLION. 273 00:10:33,520 --> 00:10:35,480 AND WE DECIDED TO GIVE IT A TRY 274 00:10:35,480 --> 00:10:36,480 AND HOPEFULLY PEOPLE WOULD HEAR 275 00:10:36,480 --> 00:10:38,960 THE STORY AND HELP US. 276 00:10:38,960 --> 00:10:41,920 OVER THE NEXT FOUR YEARS, THREE 277 00:10:41,920 --> 00:10:42,920 YEARS, WE DECIDED TO DO 278 00:10:42,920 --> 00:10:43,920 EVERYTHING WE COULD. 279 00:10:43,920 --> 00:10:45,640 TOLD THE COMMUNITY ABOUT IT. 280 00:10:45,640 --> 00:10:48,680 A WEEK LATER THEY HAD A BEER 281 00:10:48,680 --> 00:10:51,800 WORKS EVENT, MADE A BEER FOR 282 00:10:51,800 --> 00:10:53,440 HIM, $25,000 AFTER ONE WEEK OF 283 00:10:53,440 --> 00:10:55,600 COMING OUT WITH OUR STORY. 284 00:10:55,600 --> 00:11:02,680 THE GENTLEMAN ON THE LEFT, HE 285 00:11:02,680 --> 00:11:03,840 RECYCLES, EVERY MONTH I'LL BE 286 00:11:03,840 --> 00:11:04,920 HERE WITH MONTH. 287 00:11:04,920 --> 00:11:07,440 SURE ENOUGH HE SHOWS UP, WITH A 288 00:11:07,440 --> 00:11:10,760 CHECK IN HAND OR CASH, FOR THE 289 00:11:10,760 --> 00:11:11,360 LAST FOUR YEARS. 290 00:11:11,360 --> 00:11:13,720 THIS IS WHERE OUR JOURNEY BEGAN. 291 00:11:13,720 --> 00:11:15,360 I'LL PLAY A VIDEO. 292 00:11:15,360 --> 00:11:16,760 RIGHT AFTER WE CAME OUT WITH THE 293 00:11:16,760 --> 00:11:17,600 STORY THE NEWS CHANNELS PICKED 294 00:11:17,600 --> 00:11:20,400 IT UP. 295 00:11:20,400 --> 00:11:23,800 IN ONE DAY, 3 MILLION VIEWS, 296 00:11:23,800 --> 00:11:26,200 $160,000, ONE VIDEO ALONE. 297 00:11:26,200 --> 00:11:26,360 ♪ 298 00:11:26,360 --> 00:11:26,640 >> HI, BABY. 299 00:11:26,640 --> 00:11:29,360 >> WE'RE GOING TO CURE HIM. 300 00:11:29,360 --> 00:11:33,960 >> OR GIVE HIM THE BEST SHOT. 301 00:11:33,960 --> 00:11:36,560 ♪ 302 00:11:36,560 --> 00:11:42,560 303 00:11:42,560 --> 00:11:43,160 304 00:11:43,160 --> 00:11:45,960 >> A PRETTY HAPPY KID, IN 305 00:11:45,960 --> 00:11:56,120 GENERAL. 306 00:12:00,000 --> 00:12:01,800 >> MICHAEL IS A HAPPY LITTLE 307 00:12:01,800 --> 00:12:01,960 GUY. 308 00:12:01,960 --> 00:12:04,640 HE WORKS REALLY HARD TO DO BASIC 309 00:12:04,640 --> 00:12:06,080 THINGS THAT OTHER KIDS DO 310 00:12:06,080 --> 00:12:06,480 NATURALLY. 311 00:12:06,480 --> 00:12:09,120 HE NEVER GIVES UP. 312 00:12:09,120 --> 00:12:09,760 KEEPS GOING. 313 00:12:09,760 --> 00:12:13,040 HE TRIES WITH ALL HIS WILL TO DO 314 00:12:13,040 --> 00:12:16,120 SIMPLE TASKS LIKE, YOU KNOW, 315 00:12:16,120 --> 00:12:19,960 CRAWLING AND WALKING, FEEDING 316 00:12:19,960 --> 00:12:22,520 HIMSELF, THINGS WE ALL TAKE FOR 317 00:12:22,520 --> 00:12:32,680 GRANTED. 318 00:12:33,000 --> 00:12:34,240 INITIALLY IT WAS HARD. 319 00:12:34,240 --> 00:12:34,840 IT'S STILL HARD. 320 00:12:34,840 --> 00:12:38,600 EVERY DAY IS A LITTLE HARDER 321 00:12:38,600 --> 00:12:40,040 BECAUSE MICHAEL HAS NOW STARTED 322 00:12:40,040 --> 00:12:42,440 TO NOT REACH THE MILESTONES HE 323 00:12:42,440 --> 00:12:43,520 SHOULD BE REACHING. 324 00:12:43,520 --> 00:12:46,920 HE'S NOT WALKING. 325 00:12:46,920 --> 00:12:48,920 HE'S NOT TALKING. 326 00:12:48,920 --> 00:12:51,360 THAT AFFECTS YOU INTERNALLY AS A 327 00:12:51,360 --> 00:12:57,080 FAMILY. 328 00:12:57,080 --> 00:13:07,360 ♪ 329 00:13:07,360 --> 00:13:08,960 >> THERE'S NOTHING. 330 00:13:08,960 --> 00:13:12,560 THERE'S NO DRUG, THERE'S NO 331 00:13:12,560 --> 00:13:13,080 TREATMENT. 332 00:13:13,080 --> 00:13:13,800 FOR MICHAEL'S SPECIFIC DISEASE. 333 00:13:13,800 --> 00:13:15,720 >> WHEN WE WERE TOLD ABOUT 334 00:13:15,720 --> 00:13:17,720 MICHAEL'S DIAGNOSIS, THEY JUST 335 00:13:17,720 --> 00:13:21,120 SAID, GO HOME AND JUST LIVE YOUR 336 00:13:21,120 --> 00:13:21,440 LIFE. 337 00:13:21,440 --> 00:13:22,840 BUT DON'T EXPECT MUCH FROM YOUR 338 00:13:22,840 --> 00:13:23,200 SON. 339 00:13:23,200 --> 00:13:25,800 HE WILL BE IN A WHEELCHAIR, 340 00:13:25,800 --> 00:13:29,200 NON-VERBAL, HE WILL BE DELAYED. 341 00:13:29,200 --> 00:13:30,960 AND THERE'S NOTHING WE CAN DO 342 00:13:30,960 --> 00:13:31,200 FOR YOU. 343 00:13:31,200 --> 00:13:32,440 >> DOING NOTHING IS IMPOSSIBLE. 344 00:13:32,440 --> 00:13:34,640 WE CAN'T JUST SIT BACK AND SAY, 345 00:13:34,640 --> 00:13:35,760 MICHAEL CAN FALL APART AND 346 00:13:35,760 --> 00:13:36,320 THAT'S OKAY. 347 00:13:36,320 --> 00:13:38,200 THAT'S NOT OKAY FOR US. 348 00:13:38,200 --> 00:13:47,280 WE'LL DO EVERYTHING POSSIBLE. 349 00:13:47,280 --> 00:13:57,320 350 00:13:57,800 --> 00:14:00,040 >> THERE'S A PROCESS THAT COULD 351 00:14:00,040 --> 00:14:03,200 HELP MICHAEL, BUT IT INVOLVES 352 00:14:03,200 --> 00:14:06,840 THIS VERY EXPENSIVE APPROACH. 353 00:14:06,840 --> 00:14:12,840 354 00:14:12,840 --> 00:14:16,440 355 00:14:16,440 --> 00:14:18,760 >> ALZHEIMER'S DISEASE, MULTIPLE 356 00:14:18,760 --> 00:14:19,440 SCLEROSIS, PARKINSON'S DISEASE, 357 00:14:19,440 --> 00:14:21,600 ALS, AND I COULD GO ON AND ON, 358 00:14:21,600 --> 00:14:23,160 ALL THESE COMMON DISEASES ARE 359 00:14:23,160 --> 00:14:25,440 GENETIC DISEASES THAT ARE MORE 360 00:14:25,440 --> 00:14:26,680 COMPLEX, MEANING MORE THAN ONE 361 00:14:26,680 --> 00:14:28,120 GENE IS INVOLVED. 362 00:14:28,120 --> 00:14:30,160 TO FIX THOSE DISEASES, WE NEED 363 00:14:30,160 --> 00:14:32,560 TO LEARN FIRST HOW TO FIX THE 364 00:14:32,560 --> 00:14:34,360 DISEASES WHERE SINGLE GENES ARE 365 00:14:34,360 --> 00:14:36,160 DEFECTIVE SUCH AS MICHAEL'S 366 00:14:36,160 --> 00:14:37,280 DISEASE. 367 00:14:37,280 --> 00:14:43,600 IT IS THE RESEARCH IN THESE RARE 368 00:14:43,600 --> 00:14:44,400 TERRIBLE DISEASES OF CHILDREN 369 00:14:44,400 --> 00:14:45,600 THAT WILL BE THE SPRINGBOARD FOR 370 00:14:45,600 --> 00:14:48,000 US TO FIGURE OUT HOW TO TREAT 371 00:14:48,000 --> 00:14:48,920 COMMON DISEASES THAT AFFLICT 372 00:14:48,920 --> 00:14:59,240 EVERY ONE OF US. 373 00:15:00,360 --> 00:15:02,240 >> HIS PROGNOSIS IS THE FUTURE 374 00:15:02,240 --> 00:15:02,920 LOOKS BLEAK. 375 00:15:02,920 --> 00:15:06,080 HE CAN'T FIGHT FOR HIMSELF, BUT 376 00:15:06,080 --> 00:15:07,840 THAT'S WHY WE'RE FIGHTING TO DO 377 00:15:07,840 --> 00:15:09,720 IT FAST AND QUICK. 378 00:15:09,720 --> 00:15:11,680 SO WE'RE JUST HOPEFUL. 379 00:15:11,680 --> 00:15:15,280 SO HOPEFUL THAT WE MAKE IT IN 380 00:15:15,280 --> 00:15:25,400 TIME. 381 00:15:26,720 --> 00:15:28,680 >> OVER THE NEXT FOUR YEARS WE 382 00:15:28,680 --> 00:15:32,560 DID HUNDREDS OF EVENTS, GALAS, 383 00:15:32,560 --> 00:15:35,960 BALL TOURNAMENTS, NIGHTCLUBS. 384 00:15:35,960 --> 00:15:36,920 WE DID BOWL-A-THONS, MARATHONS, 385 00:15:36,920 --> 00:15:38,720 YOU NAME IT, WE DID IT. 386 00:15:38,720 --> 00:15:39,360 WE TRIED EVERYTHING HUMANLY 387 00:15:39,360 --> 00:15:42,200 POSSIBLE TO RAISE THE MONEY WE 388 00:15:42,200 --> 00:15:42,680 NEEDsED. 389 00:15:42,680 --> 00:15:44,720 WE WERE FOUND OUR COMMUNITIES 390 00:15:44,720 --> 00:15:45,600 CAME TOGETHER. 391 00:15:45,600 --> 00:15:48,360 90% WERE ORGANIZED BY SOMEONE 392 00:15:48,360 --> 00:15:49,440 ELSE, WE JUST SHOWED UP. 393 00:15:49,440 --> 00:15:51,560 THEY GAVE US THE MONEY. 394 00:15:51,560 --> 00:15:56,760 WE MOVED ON, INCREDIBLE TO HAVE 395 00:15:56,760 --> 00:15:58,440 THIS MUCH SUPPORT. 396 00:15:58,440 --> 00:15:59,640 MICHAEL WAS PROGRESSING, ALMOST 397 00:15:59,640 --> 00:16:00,400 WALKING AT THIS TIME. 398 00:16:00,400 --> 00:16:03,600 AT THE SAME TIME THE DISEASE WAS 399 00:16:03,600 --> 00:16:06,000 KICKING IN, CAUSING SEIZURES, 400 00:16:06,000 --> 00:16:08,080 UNFORTUNATELY AFTER ONE 3-HOUR 401 00:16:08,080 --> 00:16:09,480 LONG SEIZURE, HE LOST THE 402 00:16:09,480 --> 00:16:12,000 ABILITY TO BECOME VERBAL AND 403 00:16:12,000 --> 00:16:14,320 HASN'T COME BACK. 404 00:16:14,320 --> 00:16:15,160 THE COVID LOCKDOWN HAPPENED. 405 00:16:15,160 --> 00:16:20,520 WE WERE THINKING OH MY GOD, 406 00:16:20,520 --> 00:16:22,960 FORGET RAISING MONEY AND GENE 407 00:16:22,960 --> 00:16:25,120 THERAPY, HOW DO WE HAVE HIM NOT 408 00:16:25,120 --> 00:16:27,760 LOSE THE FUNCTIONS HE TRIED TO 409 00:16:27,760 --> 00:16:27,960 KEEP. 410 00:16:27,960 --> 00:16:30,680 I TRIED CALLING ALL THE POOL 411 00:16:30,680 --> 00:16:31,600 COMPANIES, I NEED MICHAEL TO 412 00:16:31,600 --> 00:16:32,680 CONTINUE THERAPY, CAN YOU 413 00:16:32,680 --> 00:16:34,440 INSTALL A POOL, I'LL PAY 414 00:16:34,440 --> 00:16:35,520 WHATEVER YOU WANT. 415 00:16:35,520 --> 00:16:37,120 THIS VIDEO IS WHAT HAPPENED THE 416 00:16:37,120 --> 00:17:24,080 WEEKEND AFTER. 417 00:17:24,080 --> 00:17:25,960 AT THE HEIGHT OF COVID THE 418 00:17:25,960 --> 00:17:27,720 COMPANY DONATED THE POOL, HIS 419 00:17:27,720 --> 00:17:29,600 HOCKEY TEAM INSTALLED IT. 420 00:17:29,600 --> 00:17:32,200 WE HAD MICHAEL SWIMMING THE NEXT 421 00:17:32,200 --> 00:17:33,000 DAY, INCREDIBLE. 422 00:17:33,000 --> 00:17:34,640 IT SHOWS YOU COMMUNITY IS REALLY 423 00:17:34,640 --> 00:17:36,720 NEEDED TO HELP, YOU KNOW, WITH 424 00:17:36,720 --> 00:17:38,000 THIS TERRIBLE DISEASE AND 425 00:17:38,000 --> 00:17:39,640 RAISING SUCH MONEY. 426 00:17:39,640 --> 00:17:40,960 WE WERE THINKING NOW WE HAVE 427 00:17:40,960 --> 00:17:43,360 MICHAEL IN THERAPY, WE HAVE TO 428 00:17:43,360 --> 00:17:44,880 RAISE MONEY AGAIN. 429 00:17:44,880 --> 00:17:47,280 AND I STARTED RIDING A BIKE, MY 430 00:17:47,280 --> 00:17:51,480 WIFE IS LIKE WHY DON'T YOU GET 431 00:17:51,480 --> 00:17:53,520 YOUR BIKE 4,000 KILOMETERS. 432 00:17:53,520 --> 00:17:57,280 HOLD ON HERE. 433 00:17:57,280 --> 00:18:00,760 I'M LYING ON THE GROUND AFTER 434 00:18:00,760 --> 00:18:00,960 TEN. 435 00:18:00,960 --> 00:18:04,360 WE GOT TO SET EXPECTATIONS 436 00:18:04,360 --> 00:18:04,680 REASONABLY. 437 00:18:04,680 --> 00:18:06,760 I SAID IF I WAS TO RIDE FROM 438 00:18:06,760 --> 00:18:08,440 TORONTO TO OTTAWA DO YOU THINK 439 00:18:08,440 --> 00:18:10,280 YOU COULD ARRANGE FOR ME TO MEET 440 00:18:10,280 --> 00:18:12,800 THE PRIME MINISTER OF CANADA? 441 00:18:12,800 --> 00:18:15,320 IF YOU DO IT, WE'LL ARRANGED. 442 00:18:15,320 --> 00:18:17,160 I TRAINED EVERY THIRD NIGHT, 443 00:18:17,160 --> 00:18:19,600 NINE TO TWO IN THE MORNING, RODE 444 00:18:19,600 --> 00:18:22,640 AS FAR AS I COULD THOSE 445 00:18:22,640 --> 00:18:22,920 EVENINGS. 446 00:18:22,920 --> 00:18:25,040 MY PARENTS AND I -- MY PARENTS 447 00:18:25,040 --> 00:18:29,480 BEHIND ME PROTECTING MOW ME SO 448 00:18:29,480 --> 00:18:31,480 HE DON'T GET RUN OVER, 400 449 00:18:31,480 --> 00:18:34,640 KILOMETERS A DAY TO GET TO 450 00:18:34,640 --> 00:18:34,920 OTTAWA. 451 00:18:34,920 --> 00:18:37,160 THAT'S MY MOM ELBOW PUMPING THE 452 00:18:37,160 --> 00:18:37,840 PRIME MINISTER. 453 00:18:37,840 --> 00:18:39,520 WE PLEADED WITH HIM NOT TO HELP 454 00:18:39,520 --> 00:18:41,840 MICHAEL BUT TO CREATE A RARE 455 00:18:41,840 --> 00:18:46,000 DISEASE CENTER OF EXCELLENCE IN 456 00:18:46,000 --> 00:18:46,240 CANADA. 457 00:18:46,240 --> 00:18:47,760 WE RAISED $168,000 DURING THAT 458 00:18:47,760 --> 00:18:48,320 ONE RIDE. 459 00:18:48,320 --> 00:18:50,320 IT WAS AN EXPERIENCE I'LL NEVER 460 00:18:50,320 --> 00:18:50,600 FORGET. 461 00:18:50,600 --> 00:18:57,200 I SHOULD HAVE KEPT RIDING 462 00:18:57,200 --> 00:18:58,840 BECAUSE I GAINED A LOT, BUT IT 463 00:18:58,840 --> 00:19:01,440 WAS A GREAT EXPERIENCE. 464 00:19:01,440 --> 00:19:03,760 AN AMAZING RESEARCHER WHO WORKS 465 00:19:03,760 --> 00:19:06,280 AT THE NIH, THE FIRST THING WAS 466 00:19:06,280 --> 00:19:09,680 SHE CAME BACK INTO THE OFFICE 467 00:19:09,680 --> 00:19:11,960 WAS TO TEST THE VECTOR ON 468 00:19:11,960 --> 00:19:12,960 MICHAEL'S CELLS. 469 00:19:12,960 --> 00:19:18,200 THIS IMAGE RIGHT HERE TO APPLY 470 00:19:18,200 --> 00:19:24,680 TO THE FDA, THIS WAS THE 471 00:19:24,680 --> 00:19:24,960 CATALYST. 472 00:19:24,960 --> 00:19:26,680 SECOND WE SHOWED IT IN CELLS WE 473 00:19:26,680 --> 00:19:28,000 COULD START TOXICOLOGY. 474 00:19:28,000 --> 00:19:29,960 THIS SHOWED THE HIGHER THE DOSE 475 00:19:29,960 --> 00:19:33,440 WE DID, THE MORE CELLS WERE ABLE 476 00:19:33,440 --> 00:19:35,520 TO RECOVER IN FIBROBLASTS. 477 00:19:35,520 --> 00:19:42,840 THIS WAS THE PRECIPICE TO MOVE 478 00:19:42,840 --> 00:19:43,720 FORWARD. 479 00:19:43,720 --> 00:19:47,240 AND THEN WILDTYPE ANIMALS WERE 480 00:19:47,240 --> 00:19:48,680 INJECTED, ONE MONTH, FIVE 481 00:19:48,680 --> 00:19:50,960 MONTHS, ONE YEAR, THE EXPRESSION 482 00:19:50,960 --> 00:19:54,000 CONTINUED AND GOT BETTER FROM 483 00:19:54,000 --> 00:19:55,760 THE TRANSGENE WE INJECTED. 484 00:19:55,760 --> 00:19:57,960 WE DID HAVE ONE PROBLEM. 485 00:19:57,960 --> 00:20:00,800 IN OUR HIGH DOSE GROUP WE HAD 486 00:20:00,800 --> 00:20:03,040 THREE ANIMALS THAT HAD ADENOMAS, 487 00:20:03,040 --> 00:20:06,960 BUT THANKFULLY ONE OF THE 488 00:20:06,960 --> 00:20:09,720 COMPANIES SHOWED THAT 53% OF 489 00:20:09,720 --> 00:20:13,240 MALE MICE 12 TO 15 MONTHS 490 00:20:13,240 --> 00:20:13,960 NATURALLY GET ADENOMAS, SO 491 00:20:13,960 --> 00:20:15,920 INSTEAD OF THE PROGRAM BEING 492 00:20:15,920 --> 00:20:20,640 OVER IT WAS JUST SOMETHING WE 493 00:20:20,640 --> 00:20:21,800 HAD TO (INDISCERNIBLE). 494 00:20:21,800 --> 00:20:23,280 THE BIG QUESTION, WE HAVE ALL 495 00:20:23,280 --> 00:20:26,960 THIS DATA NOW, DO WE MANUFACTURE 496 00:20:26,960 --> 00:20:27,960 CONCURRENTLY AND RISK 497 00:20:27,960 --> 00:20:28,320 $4.5 MILLION? 498 00:20:28,320 --> 00:20:31,800 THE ANSWER WAS YES, WE DID THAT. 499 00:20:31,800 --> 00:20:33,600 WHICH WAS CRAZY. 500 00:20:33,600 --> 00:20:36,240 WE DOSED THE RATS, DOSED 501 00:20:36,240 --> 00:20:38,840 NON-HUMAN PRIMATE, SHOWING GOOD 502 00:20:38,840 --> 00:20:39,280 DISTRIBUTION. 503 00:20:39,280 --> 00:20:48,080 WE SHOWED THE MICE WERE ABLE TO 504 00:20:48,080 --> 00:20:51,760 RECOVER HIND LEG INJECTIONS, 505 00:20:51,760 --> 00:20:56,360 AFTER TREATING MOUSE, RATS, 506 00:20:56,360 --> 00:20:57,240 NON-HUMAN PRIMATE, DOSED 507 00:20:57,240 --> 00:20:59,640 MICHAEL, THE HIGHEST DOSE EVER 508 00:20:59,640 --> 00:20:59,840 GIVEN. 509 00:20:59,840 --> 00:21:02,960 AND THANKS TO THESE AMAZING 510 00:21:02,960 --> 00:21:05,160 COMPANIES AND COLLABORATORS, 511 00:21:05,160 --> 00:21:08,320 FROM ACROSS THE WORLD, WE 512 00:21:08,320 --> 00:21:09,280 RECEIVED HEALTH CANADA APPROVAL 513 00:21:09,280 --> 00:21:10,880 DECEMBER 30, AND MY SON WAS 514 00:21:10,880 --> 00:21:12,360 DOSED ON MARCH 24 OF LAST YEAR 515 00:21:12,360 --> 00:21:14,880 WITH THE GENE THERAPY WE MADE 516 00:21:14,880 --> 00:21:15,520 FOR HIM. 517 00:21:15,520 --> 00:21:20,760 WE WEREN'T DONE. 518 00:21:20,760 --> 00:21:22,440 WE WANTED TO HELP MORE CHILDREN, 519 00:21:22,440 --> 00:21:24,920 WE HAD THREE DOSE, WE DOSED TWO 520 00:21:24,920 --> 00:21:26,680 MORE, TWO MONTHS AND FIVE MONTHS 521 00:21:26,680 --> 00:21:27,760 AGO, AT UNIVERSITY OF TEXAS 522 00:21:27,760 --> 00:21:29,200 SOUTHWESTERN MEDICAL CENTER, 523 00:21:29,200 --> 00:21:30,960 PART OF A PHASE 1/2 THAT WE 524 00:21:30,960 --> 00:21:33,920 APPLIED FOR. 525 00:21:33,920 --> 00:21:35,640 ONE THING WE REALIZED DURING 526 00:21:35,640 --> 00:21:37,080 THIS JOURNEY IS AFTER YOU 527 00:21:37,080 --> 00:21:39,040 MANUFACTURE THE DRUG AND DOSED 528 00:21:39,040 --> 00:21:40,640 PATIENTS, IT'S NOT FINANCIALLY 529 00:21:40,640 --> 00:21:42,560 VIABLE TO CONTINUE TO DO THIS. 530 00:21:42,560 --> 00:21:44,640 FOR YOU TO MAKE ONE DOSE IT 531 00:21:44,640 --> 00:21:46,680 COSTS ABOUT A MILLION DOLLARS. 532 00:21:46,680 --> 00:21:53,280 TO TREAT THAT CHILD COST COSTS 533 00:21:53,280 --> 00:21:54,360 ANOTHER MILLION CHILDREN, THE 534 00:21:54,360 --> 00:21:56,120 ONLY WAY IS TO GET THE DRUG 535 00:21:56,120 --> 00:21:58,120 APPROVED SO INSURANCE COMPANIES 536 00:21:58,120 --> 00:21:59,600 AN GOVERNMENTS WILL PAY FOR THE 537 00:21:59,600 --> 00:22:00,480 DRUG. 538 00:22:00,480 --> 00:22:01,680 IT'S SOMETHING WE REALIZED ALONG 539 00:22:01,680 --> 00:22:02,280 THIS JOURNEY. 540 00:22:02,280 --> 00:22:05,560 IF YOU LOOK AT WHAT WE DID, I'LL 541 00:22:05,560 --> 00:22:06,600 POINT OUT A COUPLE THINGS. 542 00:22:06,600 --> 00:22:09,280 FROM THE TIME WE STARTED TO THE 543 00:22:09,280 --> 00:22:10,920 TIME WE DOSED MICHAEL IN THREE 544 00:22:10,920 --> 00:22:14,880 YEARS, ONE CRAZY THING WE DID 545 00:22:14,880 --> 00:22:18,240 WAS WE HAD OUR CLINICAL BATCH 546 00:22:18,240 --> 00:22:19,440 MADE BEFORE THE NON-HUMAN TOX 547 00:22:19,440 --> 00:22:20,800 STUDY WAS FINISHED. 548 00:22:20,800 --> 00:22:24,840 IF THAT CAME BACK NEGATIVE WE 549 00:22:24,840 --> 00:22:26,560 WOULD HAVE LOST ALL 550 00:22:26,560 --> 00:22:26,880 $4.5 MILLION. 551 00:22:26,880 --> 00:22:30,320 AND IN TOTAL IT COST US 552 00:22:30,320 --> 00:22:32,360 $4.5 MILLION. 553 00:22:32,360 --> 00:22:34,320 WE WERE THANKFUL ENOUGH THAT 554 00:22:34,320 --> 00:22:35,680 JACK'S CORNER AND FOUNDATION 555 00:22:35,680 --> 00:22:36,760 COLUMBUS WAS ABLE TO PAY FOR THE 556 00:22:36,760 --> 00:22:40,800 TWO CHILDREN TO BE DOSED. 557 00:22:40,800 --> 00:22:42,000 AT UTSW. 558 00:22:42,000 --> 00:22:47,200 THIS IS OUR MASTER PLAN, WE 559 00:22:47,200 --> 00:22:57,760 DOSED MICHAEL, PART OF THE PHASE 560 00:23:05,720 --> 00:23:08,840 1/2 IN CANADA, APPLYING TO THE 561 00:23:08,840 --> 00:23:12,000 FDA FOR NATURAL HISTORY TO DO 562 00:23:12,000 --> 00:23:14,520 EIGHT PATIENTS, HOPEFULLY FIND 563 00:23:14,520 --> 00:23:18,800 EFFICACY ENDPOINT OR BIOMARKER 564 00:23:18,800 --> 00:23:19,880 APPROVAL FOR ACCELERATED 565 00:23:19,880 --> 00:23:23,920 APPROVAL BASED ON FUNDAMENTAL 566 00:23:23,920 --> 00:23:25,240 APPROVAL WE CAN'T LEAVE AN OPEN 567 00:23:25,240 --> 00:23:27,000 IND, DRUGS HAVE TO BE TAKEN ON 568 00:23:27,000 --> 00:23:29,080 OR WE'LL NEVER GET TO THE REST 569 00:23:29,080 --> 00:23:31,280 OF THE KIDS, IT'S JUST NOT 570 00:23:31,280 --> 00:23:32,120 FINANCIALLY AVAILABLE. 571 00:23:32,120 --> 00:23:35,200 THEN IT TOOK A VILLAGE OF 572 00:23:35,200 --> 00:23:36,000 PEOPLE, LITERALLY HUNDREDS OF 573 00:23:36,000 --> 00:23:37,800 PEOPLE TO DO WHAT WE DID FOR 574 00:23:37,800 --> 00:23:42,560 MICHAEL AND THESE OTHER 575 00:23:42,560 --> 00:23:42,800 CHILDREN. 576 00:23:42,800 --> 00:23:50,320 NOW AT THE NIH I'M ABLE TO THANK 577 00:23:50,320 --> 00:23:51,840 THE PEOPLE INVOLVED HERE, 578 00:23:51,840 --> 00:23:55,120 INSTRUMENTAL IN MAKING THIS WORK 579 00:23:55,120 --> 00:23:57,400 AND MAKING THIS HAPPEN. 580 00:23:57,400 --> 00:23:59,480 THE TEAM YOU SEE HERE ARE 581 00:23:59,480 --> 00:24:01,800 HELPING US MOVE THIS FORWARD OR 582 00:24:01,800 --> 00:24:03,760 ARE HELPING US WITH OUR NEW 583 00:24:03,760 --> 00:24:05,280 COMPANY WE'RE WORKING ON. 584 00:24:05,280 --> 00:24:08,320 THESE ARE HEROES, ALL THESE 585 00:24:08,320 --> 00:24:10,320 PEOPLE ON THESE IMAGES ARE 586 00:24:10,320 --> 00:24:13,520 HEROES, SAVING LIVES EVERY DAY. 587 00:24:13,520 --> 00:24:16,360 I WANT TO QUICKLY GO THROUGH HOW 588 00:24:16,360 --> 00:24:18,760 BIG THE PROBLEM IS. 589 00:24:18,760 --> 00:24:21,680 RARE DISEASES ARE HUMANITY LEVEL 590 00:24:21,680 --> 00:24:22,000 PROBLEMS. 591 00:24:22,000 --> 00:24:25,000 THERE'S MORE THAN 10,000 RARE 592 00:24:25,000 --> 00:24:26,320 DISEASES. 593 00:24:26,320 --> 00:24:27,280 THEY EFFECT MORE THAN 400 594 00:24:27,280 --> 00:24:30,040 MILLION PEOPLE, 1 IN 10 ARE 595 00:24:30,040 --> 00:24:31,240 AFFECTED. 596 00:24:31,240 --> 00:24:34,040 95% HAVE NO TREATMENT. 597 00:24:34,040 --> 00:24:36,280 AND MORE THAN 3,000 CHILDREN DIE 598 00:24:36,280 --> 00:24:37,360 EVERY SINGLE DAY. 599 00:24:37,360 --> 00:24:38,600 THIS NUMBER BLEW MY MIND. 600 00:24:38,600 --> 00:24:40,240 I CAN'T BELIEVE THAT THIS MANY 601 00:24:40,240 --> 00:24:45,240 CHILDREN ARE DYING FROM RARE 602 00:24:45,240 --> 00:24:45,800 DISEASES EVERY DAY. 603 00:24:45,800 --> 00:24:48,080 WHEN YOU LOOK AT IT, PARENTS 604 00:24:48,080 --> 00:24:49,640 LIKE ME, WE CAN'T LIVE IN FEAR. 605 00:24:49,640 --> 00:24:51,720 WE CAN'T HAVE A BAG SITTING IN 606 00:24:51,720 --> 00:24:53,040 OUR ROOM, WAITING FOR US TO RUN 607 00:24:53,040 --> 00:24:54,920 TO THE EMERGENCY ROOM BECAUSE 608 00:24:54,920 --> 00:24:57,400 OUR CHILDREN MIGHT DIE OR ARE 609 00:24:57,400 --> 00:24:59,600 GOING TO GO INTO A SEIZURE. 610 00:24:59,600 --> 00:25:01,120 WE CAN'T HAVE OUR CHILDREN 611 00:25:01,120 --> 00:25:02,440 LIVING IN PAIN, GOING THROUGH 612 00:25:02,440 --> 00:25:05,720 TESTS AND SUFFERING FROM THESE 613 00:25:05,720 --> 00:25:06,000 DISEASES. 614 00:25:06,000 --> 00:25:08,640 WE SHOULDN'T HAVE TO BURY OUR 615 00:25:08,640 --> 00:25:10,000 CHILDREN BECAUSE OF RARE 616 00:25:10,000 --> 00:25:10,320 DISEASE. 617 00:25:10,320 --> 00:25:11,880 THESE DISEASES WITH BE TREATED, 618 00:25:11,880 --> 00:25:13,160 CAN BE CURED. 619 00:25:13,160 --> 00:25:15,280 WE JUST HAVE TO FIGURE OUT A WAY 620 00:25:15,280 --> 00:25:18,200 FOR THIS FINANCIAL PROBLEM THAT 621 00:25:18,200 --> 00:25:19,640 WE HAVE. 622 00:25:19,640 --> 00:25:21,720 AND EVERY MINUTE, EVERY DAY, 623 00:25:21,720 --> 00:25:25,120 EVERY MONTH, EVERY YEAR IS LOST 624 00:25:25,120 --> 00:25:25,360 TRYING. 625 00:25:25,360 --> 00:25:26,960 FOR THE DOCTORS AND COMPANIES IN 626 00:25:26,960 --> 00:25:28,640 THE ROOM, I PLEAD WITH YOU TO 627 00:25:28,640 --> 00:25:31,120 HELP RARE DISEASES IF YOU CAN. 628 00:25:31,120 --> 00:25:32,080 HELP FAMILIES LIKE MINE. 629 00:25:32,080 --> 00:25:34,600 HELP US GET THROUGH WHAT WE'RE 630 00:25:34,600 --> 00:25:34,800 DOING. 631 00:25:34,800 --> 00:25:36,600 BECAUSE WE NEED YOUR HELP. 632 00:25:36,600 --> 00:25:37,720 WE CAN'T DO THIS ALONE. 633 00:25:37,720 --> 00:25:39,920 I NEVER DID IT ALONE, AND I HOPE 634 00:25:39,920 --> 00:25:41,640 YOU REACH OUT TO RARE DISEASE 635 00:25:41,640 --> 00:25:43,720 FAMILIES AND HELP US ALONG THIS 636 00:25:43,720 --> 00:25:43,960 JOURNEY. 637 00:25:43,960 --> 00:25:48,440 THAT'S THE MAIN REASON I STARTED 638 00:25:48,440 --> 00:25:49,280 ELPIDA THERAPEUTICS, I REALIZED 639 00:25:49,280 --> 00:25:51,840 I WANTED TO MAKE A SMALL DENT, 640 00:25:51,840 --> 00:25:53,360 TO TRY TO TREAT THESE DISEASES, 641 00:25:53,360 --> 00:25:56,440 GET THEM APPROVED. 642 00:25:56,440 --> 00:26:00,800 WE STARTED IN MAY, WE WERE THEN 643 00:26:00,800 --> 00:26:03,360 CREATING A PARTNERSHIP, THE GOAL 644 00:26:03,360 --> 00:26:06,600 TO TAKE PROGRAMS 645 00:26:06,600 --> 00:26:07,200 NON-COMMERCIALLY VIABLE, LESS 646 00:26:07,200 --> 00:26:09,560 THAN 300 IN THE WORLD, FROM 647 00:26:09,560 --> 00:26:12,080 SELECTION TO BLA, GET THE DRUG 648 00:26:12,080 --> 00:26:15,640 APPROVED IN THE HANDS OF 649 00:26:15,640 --> 00:26:22,920 CORPORATIONS LIKES ROCHE, 650 00:26:22,920 --> 00:26:24,520 NOVARTIS, PFIZER, TO DO MORE 651 00:26:24,520 --> 00:26:25,120 PROGRAMS. 652 00:26:25,120 --> 00:26:32,760 WE'VE TAKEN ON TWO PROGRAMS. 653 00:26:32,760 --> 00:26:35,920 SPG-50, MY SON, CMT4J, AND IN 654 00:26:35,920 --> 00:26:43,040 CONTRACT TO TAKE ON THREE MORE, 655 00:26:43,040 --> 00:26:45,440 TO GET THESE TREATED BY 2025, TO 656 00:26:45,440 --> 00:26:47,200 GET AN ENDPOINT AND SHOW 657 00:26:47,200 --> 00:26:48,400 CHILDREN IMPROVE, WE'LL BE ABLE 658 00:26:48,400 --> 00:26:50,360 TO CHANGE THEIR LIVES. 659 00:26:50,360 --> 00:26:55,840 AND I WANT TO THANK THE BGTC FOR 660 00:26:55,840 --> 00:26:57,920 BELIEVING IN US, A SMALL 661 00:26:57,920 --> 00:26:59,880 COMPANY, ONE PERSON AT THE TIME, 662 00:26:59,880 --> 00:27:00,960 TO MOVE FORWARD AND HELP THESE 663 00:27:00,960 --> 00:27:04,480 CHILDREN WITH THESE RARE 664 00:27:04,480 --> 00:27:04,840 DISEASES. 665 00:27:04,840 --> 00:27:07,080 THANK YOU. 666 00:27:07,080 --> 00:27:13,160 667 00:27:13,160 --> 00:27:14,040 668 00:27:14,040 --> 00:27:15,120 I THINK WE'RE OPEN FOR 669 00:27:15,120 --> 00:27:16,840 QUESTIONS. 670 00:27:16,840 --> 00:27:19,400 IF ANYBODY HAS ANY. 671 00:27:19,400 --> 00:27:20,560 HOW IS MICHAEL? 672 00:27:20,560 --> 00:27:21,440 HE'S -- IT'S INTERESTING. 673 00:27:21,440 --> 00:27:23,520 HE WAS DOING NINE WEEKS OF 674 00:27:23,520 --> 00:27:26,840 INTENSE THERAPY AT A HOSPITAL IN 675 00:27:26,840 --> 00:27:27,120 CANADA. 676 00:27:27,120 --> 00:27:30,000 AND THEN WE WERE -- HE GOT SICK. 677 00:27:30,000 --> 00:27:33,720 WE WERE AT THE HOSPITAL, HE GOT 678 00:27:33,720 --> 00:27:34,920 C. DIFFICILE, IT'S A TERRIBLE 679 00:27:34,920 --> 00:27:36,240 STOMACH INFECTION. 680 00:27:36,240 --> 00:27:37,240 HE LOST TEN POUNDS, COULDN'T GO 681 00:27:37,240 --> 00:27:40,880 TO SCHOOL FOR SIX MONTHS, SOME 682 00:27:40,880 --> 00:27:42,360 DAYS COULDN'T EVEN GET OUT OF 683 00:27:42,360 --> 00:27:42,560 BED. 684 00:27:42,560 --> 00:27:46,680 WE HAD TO CARRY HIM DOWNSTAIRS. 685 00:27:46,680 --> 00:27:49,840 HE'S JUST GETTING BACK TO WHERE 686 00:27:49,840 --> 00:27:51,480 HE WAS BEFORE, COGNITION 687 00:27:51,480 --> 00:27:52,800 SIGNIFICANTLY IMPROVE, ABLE TO 688 00:27:52,800 --> 00:27:53,880 COMMUNICATE MUCH BETTER. 689 00:27:53,880 --> 00:27:56,080 HIS MOBILITY, WHERE HE WOULD 690 00:27:56,080 --> 00:27:57,160 HAVE BEEN WALKING, UNFORTUNATELY 691 00:27:57,160 --> 00:27:59,880 HE'S JUST CATCHING UP NOW. 692 00:27:59,880 --> 00:28:02,200 THE OTHER TWO CHILDREN ARE DOING 693 00:28:02,200 --> 00:28:03,520 AMAZING, AND WE'RE HAPPY WITH 694 00:28:03,520 --> 00:28:06,360 THE RESULTS SO FAR. 695 00:28:06,360 --> 00:28:08,920 696 00:28:08,920 --> 00:28:17,320 >> QUESTIONS? 697 00:28:17,320 --> 00:28:19,720 698 00:28:19,720 --> 00:28:21,400 >> HI. 699 00:28:21,400 --> 00:28:22,120 GREAT TALK. 700 00:28:22,120 --> 00:28:23,880 >> THANK YOU. 701 00:28:23,880 --> 00:28:25,320 >> SO, MICHAEL'S CONDITION IS 702 00:28:25,320 --> 00:28:28,920 REALLY ULTRA RARE, IS THERE A 703 00:28:28,920 --> 00:28:32,200 THRESHOLD FOR WHEN A CONDITION 704 00:28:32,200 --> 00:28:36,120 BECOMES MORE FINANCIALLY VIABLE? 705 00:28:36,120 --> 00:28:38,320 >> YEAH, THE NUMBER IS AROUND 706 00:28:38,320 --> 00:28:40,400 2,000 PATIENTS WORLDWIDE. 707 00:28:40,400 --> 00:28:41,320 SO THERE'S PROBABLY A FEW 708 00:28:41,320 --> 00:28:44,040 THOUSAND DISEASES THAT FALL 709 00:28:44,040 --> 00:28:49,080 UNDER THAT THRESHOLD. 710 00:28:49,080 --> 00:28:49,640 PERFECT. 711 00:28:49,640 --> 00:28:53,920 WELL, IF THERE'S NO MORE 712 00:28:53,920 --> 00:28:55,000 QUESTIONS WE'LL INTRODUCE RON 713 00:28:55,000 --> 00:28:56,240 FOR THE NEXT TALK. 714 00:28:56,240 --> 00:29:07,160 >> I'M STILL RON BARTEK, FARA, 715 00:29:07,160 --> 00:29:09,240 MY WIFE AND I FOUNDED 25 YEARS 716 00:29:09,240 --> 00:29:11,200 AGO WHEN OUR SON KEITH WAS 717 00:29:11,200 --> 00:29:12,640 DIAGNOSED -- IT'S HARD TO SAY 25 718 00:29:12,640 --> 00:29:14,360 YEARS AGO. 719 00:29:14,360 --> 00:29:15,320 I'M GOING TO BE BRIEF AND GET 720 00:29:15,320 --> 00:29:18,000 OUT OF THE WAY OF THESE 721 00:29:18,000 --> 00:29:20,480 WONDERFUL PANELISTS FOR A 722 00:29:20,480 --> 00:29:22,120 WONDERFUL DISCUSSION OF THE 723 00:29:22,120 --> 00:29:25,400 THING THAT TERRY SO INSPIRINGLY 724 00:29:25,400 --> 00:29:27,480 INTRODUCED, THAT OF GENE 725 00:29:27,480 --> 00:29:27,840 THERAPY. 726 00:29:27,840 --> 00:29:32,720 JUST A BIT ABOUT MY BACKGROUND. 727 00:29:32,720 --> 00:29:35,360 MY WIFE AND I BEGAN 728 00:29:35,360 --> 00:29:36,560 COLLABORATING WITH, WORKING WITH 729 00:29:36,560 --> 00:29:46,080 NINDS, EVEN BEFORE WE 730 00:29:46,080 --> 00:29:47,240 ESTABLISHED FARA, MET OUR 731 00:29:47,240 --> 00:29:49,040 PROGRAM DIRECTOR 26 YEARS AGO, 732 00:29:49,040 --> 00:29:50,600 BEGINNING OUR PROCESS WITH THIS 733 00:29:50,600 --> 00:29:52,640 WONDERFUL INSTITUTE. 734 00:29:52,640 --> 00:29:56,720 I WAS ABLE TO -- A PROGRAM 735 00:29:56,720 --> 00:30:00,320 DIRECTOR TOOK US TO OUR FIRST 736 00:30:00,320 --> 00:30:01,000 NATIONAL ADVISORY COUNCIL 737 00:30:01,000 --> 00:30:03,920 MEETING IN THE MAIN BUILDING, 738 00:30:03,920 --> 00:30:05,520 AND THAT STARTED THE BALL 739 00:30:05,520 --> 00:30:06,960 ROLLING OF A WONDERFUL 740 00:30:06,960 --> 00:30:10,320 COLLABORATION OVER THOSE 25 741 00:30:10,320 --> 00:30:12,280 YEARS. 742 00:30:12,280 --> 00:30:13,920 CULMINATING -- NOT REALLY 743 00:30:13,920 --> 00:30:16,560 CULMINATING, BUT INCLUDING A 744 00:30:16,560 --> 00:30:18,200 FOUR-YEAR STINT ON THE NATIONAL 745 00:30:18,200 --> 00:30:20,760 ADVISORY COUNCIL OF NINDS. 746 00:30:20,760 --> 00:30:22,680 AND REALLY PLEASED TO SAY ALSO 747 00:30:22,680 --> 00:30:24,600 GOT THE OPPORTUNITY TO SERVE ON 748 00:30:24,600 --> 00:30:30,120 THE NATIONAL ADVISORY COUNCIL OF 749 00:30:30,120 --> 00:30:30,880 NATIONAL CENTER FOR NCATS, AND 750 00:30:30,880 --> 00:30:33,680 THIS SOURCE OF ONE OF OUR 751 00:30:33,680 --> 00:30:36,640 PANELISTS, P.J. BROOKS, IS A KEY 752 00:30:36,640 --> 00:30:40,760 MEMBER OF THAT TEAM. 753 00:30:40,760 --> 00:30:44,560 ALL THAT COLLABORATION, YOU 754 00:30:44,560 --> 00:30:47,560 KNOW, HAS REALLY STIMULATED OUR 755 00:30:47,560 --> 00:30:50,720 RESEARCH IN OUR RARE DISEASE, A 756 00:30:50,720 --> 00:30:52,000 RARE NEUROMUSCULAR DISEASE. 757 00:30:52,000 --> 00:30:54,440 I'LL SAY THAT OUR DEEP 758 00:30:54,440 --> 00:30:57,880 INVOLVEMENT WITH NINDS HAS 759 00:30:57,880 --> 00:31:00,440 PROVEN TO BE ABSOLUTELY 760 00:31:00,440 --> 00:31:02,800 BENEFICIAL TO OUR SCIENTISTS. 761 00:31:02,800 --> 00:31:06,280 ONCE WE STARTED SEARCHING AS A 762 00:31:06,280 --> 00:31:09,560 TWO-WAY COMMUNICATOR BETWEEN 763 00:31:09,560 --> 00:31:11,760 NINDS AND OUR SCIENTISTS, 764 00:31:11,760 --> 00:31:13,200 INFORMING OUR SCIENTISTS OF 765 00:31:13,200 --> 00:31:15,600 OPPORTUNITIES HERE AT THE 766 00:31:15,600 --> 00:31:16,120 INSTITUTE, INFORMING THE 767 00:31:16,120 --> 00:31:19,120 INSTITUTE OF THE WORK GOING ON 768 00:31:19,120 --> 00:31:22,600 AMONG OUR SCIENTISTS, YOU CAN 769 00:31:22,600 --> 00:31:25,040 IMAGINE HOW SIGNIFICANTLY ROSE 770 00:31:25,040 --> 00:31:27,880 THE NUMBER OF NIH GRANTS TO OUR 771 00:31:27,880 --> 00:31:29,960 RESEARCH SCIENTISTS. 772 00:31:29,960 --> 00:31:34,320 I JUST COULDN'T ENCOURAGE YOU 773 00:31:34,320 --> 00:31:36,200 MORE TO USE THESE OPPORTUNITIES 774 00:31:36,200 --> 00:31:38,720 TO MEET YOUR PROGRAM DIRECTORS 775 00:31:38,720 --> 00:31:39,600 AS WE DID. 776 00:31:39,600 --> 00:31:40,760 SOME OF YOU HAVE ALREADY SPENT 777 00:31:40,760 --> 00:31:42,000 TIME THIS MORNING WITH YOUR 778 00:31:42,000 --> 00:31:43,960 PROGRAM DIRECTORS. 779 00:31:43,960 --> 00:31:45,520 IF YOU HAVEN'T DONE THAT, PLEASE 780 00:31:45,520 --> 00:31:48,360 DO IT. 781 00:31:48,360 --> 00:31:49,120 IT'S A PHENOMENALLY IMPORTANT 782 00:31:49,120 --> 00:31:52,960 AND GOOD WAY TO GET TO KNOW THE 783 00:31:52,960 --> 00:31:56,000 WORKINGS OF THE INSTITUTE AND 784 00:31:56,000 --> 00:31:58,200 COMMUNICATE THOSE WORKINGS TO 785 00:31:58,200 --> 00:32:00,720 YOUR WHOLE COMMUNITY, YOUR 786 00:32:00,720 --> 00:32:03,360 PATIENT FAMILIES, YOUR 787 00:32:03,360 --> 00:32:04,520 SCIENTISTS, AND SO FORTH. 788 00:32:04,520 --> 00:32:08,440 I'D LIKE TO TAKE A MOMENT HERE. 789 00:32:08,440 --> 00:32:09,640 I GET EMOTIONAL. 790 00:32:09,640 --> 00:32:11,520 I'M SORRY. 791 00:32:11,520 --> 00:32:15,080 TO APPLAUD THE NINDS FOR ITS 792 00:32:15,080 --> 00:32:16,040 TREMENDOUS DEVOTION TO ALL OF US 793 00:32:16,040 --> 00:32:17,680 AND THE WORK THAT THEY DO FOR 794 00:32:17,680 --> 00:32:19,400 ALL OF OUR RARE DISEASE 795 00:32:19,400 --> 00:32:28,040 PATIENTS. 796 00:32:28,040 --> 00:32:34,000 NOW I WILL TRY TO SOBER UP AND 797 00:32:34,000 --> 00:32:36,480 TURN TO THIS WONDERFUL PANEL. 798 00:32:36,480 --> 00:32:39,320 OUR INTENTION IS TO EXPAND ON 799 00:32:39,320 --> 00:32:42,520 THE PARTNERSHIPS AND 800 00:32:42,520 --> 00:32:44,120 COLLABORATIONS THAT TERRY 801 00:32:44,120 --> 00:32:45,480 INTRODUCED, IN ADVANTAGING THE 802 00:32:45,480 --> 00:32:47,080 WORK IN THIS VERY IMPORTANT 803 00:32:47,080 --> 00:32:48,720 FIELD OF GENE THERAPY, IMPORTANT 804 00:32:48,720 --> 00:32:56,600 TO I THINK EVERYBODY IN THE ROOM 805 00:32:56,600 --> 00:32:59,960 AND ONLINE, WITH EMPHASIS ON THE 806 00:32:59,960 --> 00:33:04,200 ROLE PATIENTS AND ADVOCATES 807 00:33:04,200 --> 00:33:07,560 PLAY, AND PLAY MORE PROMINENTLY. 808 00:33:07,560 --> 00:33:10,840 I'M GOING TO KICK OFF BY 809 00:33:10,840 --> 00:33:11,920 INTRODUCING AND EACH WILL 810 00:33:11,920 --> 00:33:13,040 INTRODUCE THEMSELVES. 811 00:33:13,040 --> 00:33:15,200 BUT I WANT TO INTRODUCE LEAD-OFF 812 00:33:15,200 --> 00:33:17,760 HITTER, I'M A BASEBALL FAN. 813 00:33:17,760 --> 00:33:20,280 DR. COURTNEY SILVERTHORN. 814 00:33:20,280 --> 00:33:22,160 >>NINDS IS AN EARLY AND STRONG 815 00:33:22,160 --> 00:33:25,880 SUPPORTER OF THE WORK OF THE 816 00:33:25,880 --> 00:33:27,840 BESPOKE GENE CONSORTIUM, REALLY 817 00:33:27,840 --> 00:33:29,480 THE GOAL TO STREAMLINE THE 818 00:33:29,480 --> 00:33:32,560 PROCESS OF BRINGING SOMETHING 819 00:33:32,560 --> 00:33:34,080 FORWARDS THROUGH PRE-CLINICAL 820 00:33:34,080 --> 00:33:36,560 TESTING INTO A FIRST-IN-HUMAN 821 00:33:36,560 --> 00:33:36,960 CLINICAL TRIAL. 822 00:33:36,960 --> 00:33:40,440 AND SO WE HAVE BEEN ON AN 823 00:33:40,440 --> 00:33:42,480 18-MONTH JOURNEY TO IDENTIFY 824 00:33:42,480 --> 00:33:44,160 PROMISING CANDIDATES FOR 825 00:33:44,160 --> 00:33:45,880 CLINICAL TRIAL APPLICATIONS, HAD 826 00:33:45,880 --> 00:33:51,040 A VERY OPEN PROCESS THROUGHOUT 827 00:33:51,040 --> 00:33:54,440 2021 INTO 2022, TO IDENTIFY WHAT 828 00:33:54,440 --> 00:33:55,320 ULTIMATELY LANDED ON EIGHT 829 00:33:55,320 --> 00:33:58,520 CLINICAL TRIALS IN EIGHT RARE 830 00:33:58,520 --> 00:34:00,920 DISEASES WITH THE GOAL OF USING 831 00:34:00,920 --> 00:34:02,800 THESE AS TEST CASES TO MOVE THE 832 00:34:02,800 --> 00:34:03,560 ENTIRE FIELD FORWARD. 833 00:34:03,560 --> 00:34:04,640 IF YOU TAKE NOTHING ELSE HOME 834 00:34:04,640 --> 00:34:08,640 FROM THE MESSAGE OF THE BGTC, WE 835 00:34:08,640 --> 00:34:14,440 HAVE 8 DISEASES IN THE PORTFOLO 836 00:34:14,440 --> 00:34:16,800 WE WANT TO HELP EVERY GENE 837 00:34:16,800 --> 00:34:19,520 THERAPY THAT COULD MOVE FORWARD 838 00:34:19,520 --> 00:34:23,520 IN THIS SPACE BY PROVIDING 839 00:34:23,520 --> 00:34:24,040 MANUFACTURERS PROTOCOLS, 840 00:34:24,040 --> 00:34:24,680 REGULATORY TEMPLATES, RESOURCES 841 00:34:24,680 --> 00:34:26,920 PEOPLE NEED TO HAVE THE MORE 842 00:34:26,920 --> 00:34:29,760 STREAMLINED PATH FORWARD INTO 843 00:34:29,760 --> 00:34:32,040 THEIR JOURNEY AND INTO THEIR 844 00:34:32,040 --> 00:34:35,040 TREATMENTS FOR THEIR CHILDREN. 845 00:34:35,040 --> 00:34:37,080 THE BESPOKE CONSORTIUM IS PART 846 00:34:37,080 --> 00:34:38,400 OF THE ACCELERATED MEDICINES 847 00:34:38,400 --> 00:34:39,200 PARTNERSHIP PROGRAM. 848 00:34:39,200 --> 00:34:41,240 THIS DATES BACK TO 2014 WITH THE 849 00:34:41,240 --> 00:34:43,640 FOUNDATION OF THREE PROJECTS, 850 00:34:43,640 --> 00:34:49,480 THREE DISEASE AREAS, FOCUSED ON 851 00:34:49,480 --> 00:34:50,120 PRE-COMPETITIVE ADVANCEMENTS. 852 00:34:50,120 --> 00:34:51,880 IN GENE THERAPY THE AREA WE 853 00:34:51,880 --> 00:34:53,320 IDENTIFIED WAS ABOUT THE PROCESS 854 00:34:53,320 --> 00:34:56,280 AND THE PROCESS OF THE PLATFORM. 855 00:34:56,280 --> 00:35:01,000 THAT WAS WHERE WE FOCUSED OUR 856 00:35:01,000 --> 00:35:01,360 EFFORTS. 857 00:35:01,360 --> 00:35:07,400 WE'RE THE ONLY PROGRAM THAT IS 858 00:35:07,400 --> 00:35:10,080 IN DISEASE AGNOSTIC STATES, 859 00:35:10,080 --> 00:35:11,080 FOCUSED ON THAT PROCESS RATHER 860 00:35:11,080 --> 00:35:12,600 THAN DISEASE OR DISEASE AREA. 861 00:35:12,600 --> 00:35:14,440 MY BACKGROUND IS NOT GENE 862 00:35:14,440 --> 00:35:15,880 THERAPY, IT'S IN PHARMACOLOGY 863 00:35:15,880 --> 00:35:18,960 WHICH I HAVE NOT USED SINCE 864 00:35:18,960 --> 00:35:21,080 2009, MY EXPERTISE IS IN 865 00:35:21,080 --> 00:35:22,360 PUBLIC/PRIVATE PARTNERSHIPS AND 866 00:35:22,360 --> 00:35:24,280 I'VE BEEN GIVEN THIS INCREDIBLE 867 00:35:24,280 --> 00:35:25,440 OPPORTUNITY TO SHEPHERD THE WORK 868 00:35:25,440 --> 00:35:29,360 THAT SO MANY PEOPLE ARE DOING. 869 00:35:29,360 --> 00:35:30,760 I LOOKED ACROSS OUR STEERING 870 00:35:30,760 --> 00:35:33,320 COMMITTEES AND OUR WORKING 871 00:35:33,320 --> 00:35:34,520 GROUPS, AND OUR ACADEMIC 872 00:35:34,520 --> 00:35:35,520 INSTITUTIONS THAT ARE RECEIVING 873 00:35:35,520 --> 00:35:38,200 FUNDING FROM THE PROGRAM. 874 00:35:38,200 --> 00:35:39,360 IT'S WELL OVER 200 INDIVIDUALS 875 00:35:39,360 --> 00:35:41,200 WHO ARE CONTRIBUTING TO THE 876 00:35:41,200 --> 00:35:41,520 PROCESS. 877 00:35:41,520 --> 00:35:44,360 SO IT'S JUST SUCH AN OPPORTUNITY 878 00:35:44,360 --> 00:35:46,760 TO BE PART OF THAT AND TO 879 00:35:46,760 --> 00:35:49,080 OVERSEE THE PROGRESS THAT WE'RE 880 00:35:49,080 --> 00:35:51,040 STARTING TO MAKE. 881 00:35:51,040 --> 00:35:52,800 REALLY EXCITED TO DISCUSS MORE 882 00:35:52,800 --> 00:35:53,000 TODAY. 883 00:35:53,000 --> 00:35:55,760 >> THANK YOU VERY MUCH, DR. 884 00:35:55,760 --> 00:35:57,520 SILVER THORN. 885 00:35:57,520 --> 00:36:03,120 BATTING SECOND IS COMING TO US 886 00:36:03,120 --> 00:36:04,680 VIRTUALLY, DR. P.J. BROOKS. 887 00:36:04,680 --> 00:36:06,160 ARE YOU THERE? 888 00:36:06,160 --> 00:36:08,120 >> GOOD MORNING, EVERYBODY. 889 00:36:08,120 --> 00:36:09,800 CAN YOU SEE ME AND HEAR ME? 890 00:36:09,800 --> 00:36:14,160 >> WE CAN HEAR YOU. 891 00:36:14,160 --> 00:36:18,000 WE CANNOT YET SEE YOU. 892 00:36:18,000 --> 00:36:21,520 893 00:36:21,520 --> 00:36:27,360 >> HOW ABOUT NOW? 894 00:36:27,360 --> 00:36:29,960 >> THAT WAS WALTER BY THE WAY, 895 00:36:29,960 --> 00:36:30,120 P.J. 896 00:36:30,120 --> 00:36:30,720 >> OKAY. 897 00:36:30,720 --> 00:36:35,920 NOW YOU CAN SEE ME? 898 00:36:35,920 --> 00:36:37,400 899 00:36:37,400 --> 00:36:39,800 SO, YEAH, HAPPY TO BE HERE. 900 00:36:39,800 --> 00:36:41,760 AND SORRY I CAN'T BE THERE IN 901 00:36:41,760 --> 00:36:42,000 PERSON. 902 00:36:42,000 --> 00:36:44,760 I HAD ANOTHER COMMITMENT. 903 00:36:44,760 --> 00:36:46,080 BUT HAPPY TO PARTICIPATE. 904 00:36:46,080 --> 00:36:47,720 SO I WANTED TO TELL YOU ABOUT 905 00:36:47,720 --> 00:36:51,560 SOME THINGS WE'RE DOING AT 906 00:36:51,560 --> 00:36:51,840 NCATS. 907 00:36:51,840 --> 00:36:55,720 AT NCATS, WE TRY TO FOCUS ON 908 00:36:55,720 --> 00:36:59,640 COMMONALITIES ACROSS MULTIPLE 909 00:36:59,640 --> 00:36:59,920 DISEASES. 910 00:36:59,920 --> 00:37:00,920 AND THAT SEEMS LIKE THE MOST 911 00:37:00,920 --> 00:37:03,160 PRACTICAL WAY TO DEAL WITH THE 912 00:37:03,160 --> 00:37:05,560 THOUSANDS OF RARE DISEASES AND 913 00:37:05,560 --> 00:37:09,520 PARTICULARLY THE RARE GENETIC 914 00:37:09,520 --> 00:37:12,760 DISEASES. 915 00:37:12,760 --> 00:37:15,600 AND SO WE HAVE -- ARE INVOLVED 916 00:37:15,600 --> 00:37:17,800 IN MULTIPLE PROGRAMS TO DO THIS, 917 00:37:17,800 --> 00:37:22,000 ONE IS THE BESPOKE GENE THERAPY 918 00:37:22,000 --> 00:37:24,280 CONSORTIUM YOU HEARD ABOUT FROM 919 00:37:24,280 --> 00:37:24,640 COURTNEY. 920 00:37:24,640 --> 00:37:26,360 ANOTHER ACTIVITY THAT WE HAVE 921 00:37:26,360 --> 00:37:30,200 GOING ON AND WE BEGAN THIS 922 00:37:30,200 --> 00:37:33,800 BEFORE THE BGTC, THE PAVE GT 923 00:37:33,800 --> 00:37:35,320 PROGRAM, PLATFORM VECTOR GENE 924 00:37:35,320 --> 00:37:35,600 THERAPIES. 925 00:37:35,600 --> 00:37:40,360 THE IDEA OF THIS ONE IS IF WE 926 00:37:40,360 --> 00:37:42,720 CAN USE THE SAME AAV VECTOR FOR 927 00:37:42,720 --> 00:37:43,560 MULTIPLE DISEASES AND KEEP 928 00:37:43,560 --> 00:37:46,960 EVERYTHING THE SAME EXCEPT FOR 929 00:37:46,960 --> 00:37:47,960 THE THERAPEUTIC INSERT, THAT WE 930 00:37:47,960 --> 00:37:52,040 OUGHT TO BE ABLE TO IDENTIFY 931 00:37:52,040 --> 00:37:54,960 MORE STREAMLINED WAYS OF 932 00:37:54,960 --> 00:37:56,160 STARTING UP CLINICAL TRIALS AND 933 00:37:56,160 --> 00:37:59,200 TO DO THAT WE'VE SELECTED FOUR 934 00:37:59,200 --> 00:38:01,080 DIFFERENT DISEASES, ALL DISEASES 935 00:38:01,080 --> 00:38:03,240 HAVE NO COMMERCIAL INTEREST, AND 936 00:38:03,240 --> 00:38:04,600 ALL DISEASES UNDER STUDY AT THE 937 00:38:04,600 --> 00:38:07,960 NIH CLINICAL CENTER. 938 00:38:07,960 --> 00:38:10,640 TWO OF THESE ARE METABOLIC 939 00:38:10,640 --> 00:38:11,520 DISEASES. 940 00:38:11,520 --> 00:38:12,800 AND TWO ARE NEUROMUSCULAR 941 00:38:12,800 --> 00:38:13,880 JUNCTION DISEASES. 942 00:38:13,880 --> 00:38:16,520 AND THE IDEA IS THAT WOULD KEEP 943 00:38:16,520 --> 00:38:18,920 EVERYTHING THE SAME, EXCEPT THE 944 00:38:18,920 --> 00:38:19,600 THERAPEUTIC INSERT. 945 00:38:19,600 --> 00:38:23,400 WE WOULD PROPOSE TO THE FDA 946 00:38:23,400 --> 00:38:25,360 CERTAIN WAYS OF STREAMLINING THE 947 00:38:25,360 --> 00:38:25,600 PROCESS. 948 00:38:25,600 --> 00:38:28,440 THEY WOULD RESPOND TO US IN THE 949 00:38:28,440 --> 00:38:29,520 INTERACT AND PRE-IND MEETINGS, 950 00:38:29,520 --> 00:38:32,040 AND WE WOULD MAKE THIS 951 00:38:32,040 --> 00:38:32,600 INFORMATION PUBLIC. 952 00:38:32,600 --> 00:38:33,920 R. SO THAT EVERYBODY CAN LEARN 953 00:38:33,920 --> 00:38:37,320 FROM US AS WE GO THROUGH THE 954 00:38:37,320 --> 00:38:37,640 PROCESS. 955 00:38:37,640 --> 00:38:44,240 AND ONE OF OUR PARTNERS IN THIS 956 00:38:44,240 --> 00:38:47,600 IS DR. CARSON BONNEMAN FROM 957 00:38:47,600 --> 00:38:50,280 NINDS, TWO STUDIES NEUROMUSCULAR 958 00:38:50,280 --> 00:38:50,560 DISEASES. 959 00:38:50,560 --> 00:38:56,160 WE'VE MADE THIS WORK PUBLIC, 960 00:38:56,160 --> 00:38:57,480 RELEASED ORPHAN PRODUCTS DRUG 961 00:38:57,480 --> 00:39:05,920 DESIGNATION WE FILED FOR A FIRST 962 00:39:05,920 --> 00:39:08,560 DISEASES PROPRIONIC, YOU CAN 963 00:39:08,560 --> 00:39:10,520 DOWNLOAD THAT AND FIND THE 964 00:39:10,520 --> 00:39:11,640 PRODUCT TO DEVELOP THE ORPHAN 965 00:39:11,640 --> 00:39:11,880 PRODUCTS. 966 00:39:11,880 --> 00:39:13,280 THESE ARE THE THINGS WE'RE DOING 967 00:39:13,280 --> 00:39:18,600 IN THE GENE THERAPY SPACE. 968 00:39:18,600 --> 00:39:21,120 ALONG WITH THE BGTC, OF COURSE. 969 00:39:21,120 --> 00:39:26,680 IN ADDITION TO GENE THERAPY WE 970 00:39:26,680 --> 00:39:28,080 INTERESTED IN GENOME EDITING, 971 00:39:28,080 --> 00:39:31,920 PERHAPS MORE BROADLY APPLICABLE 972 00:39:31,920 --> 00:39:32,760 PLATFORM FOR GENETIC DISEASES. 973 00:39:32,760 --> 00:39:37,400 I'M ONE OF THE CO-COORDINATORS 974 00:39:37,400 --> 00:39:39,760 WITH TIM LAVOTE FROM NINDS OF 975 00:39:39,760 --> 00:39:41,760 THE SOMATIC CELL GENOME EDITING 976 00:39:41,760 --> 00:39:44,400 PROGRAM, FUNDED BY THE NIH 977 00:39:44,400 --> 00:39:45,240 COMMON FUND. 978 00:39:45,240 --> 00:39:49,120 WE'RE NOW ABOUT TO START OUR 979 00:39:49,120 --> 00:39:50,440 SECOND YEAR, SECOND PHASE. 980 00:39:50,440 --> 00:39:53,880 THE FIRST PHASE WAS FOCUSED ON 981 00:39:53,880 --> 00:39:55,360 THE DEVELOPMENT OF TECHNOLOGIES, 982 00:39:55,360 --> 00:39:58,480 AND TOOLS TO ACCELERATE GENOME 983 00:39:58,480 --> 00:39:59,760 EDITING INTO THE CLINIC. 984 00:39:59,760 --> 00:40:03,480 WE'RE NOW IN THE SECOND PHASE, 985 00:40:03,480 --> 00:40:10,600 FOCUSED MORE ON GETTING INTO THE 986 00:40:10,600 --> 00:40:14,240 CLINIC, AND GETTING APPROVED 987 00:40:14,240 --> 00:40:16,480 INDs TO MAKE AVAILABLE TO 988 00:40:16,480 --> 00:40:17,400 OTHERS SHOULD THEY CHOOSE TO 989 00:40:17,400 --> 00:40:19,240 PICK UP AND EVEN SUPPORTING 990 00:40:19,240 --> 00:40:21,520 CLINICAL TRIALS OF GENOME 991 00:40:21,520 --> 00:40:22,480 EDITORS WITH REQUIREMENTS THAT 992 00:40:22,480 --> 00:40:27,120 THESE ARE DONE AS THERAPEUTIC 993 00:40:27,120 --> 00:40:27,920 PLATFORM FOR MULTIPLE DISEASES, 994 00:40:27,920 --> 00:40:31,600 SOCK WE THINK IS VERY IMPORTANT. 995 00:40:31,600 --> 00:40:34,160 WE TRIED TO DEVELOP THERAPEUTIC 996 00:40:34,160 --> 00:40:35,560 PLATFORMS AS THERAPEUTIC 997 00:40:35,560 --> 00:40:36,320 PLATFORMS FROM THE BEGINNING, 998 00:40:36,320 --> 00:40:38,840 AND TO GET AWAY FROM THE IDEA OF 999 00:40:38,840 --> 00:40:40,040 DEVELOPING ONE DRUG FOR ONE 1000 00:40:40,040 --> 00:40:41,600 DISEASE THAT IS JUST GOING TO 1001 00:40:41,600 --> 00:40:42,920 TAKE FAR TOO LONG GIVEN THE 1002 00:40:42,920 --> 00:40:46,320 AMOUNT OF DISEASES THAT WE HAVE 1003 00:40:46,320 --> 00:40:47,840 TO DEAL WITH. 1004 00:40:47,840 --> 00:40:50,160 AND FINALLY THE GENOME EDITING 1005 00:40:50,160 --> 00:40:51,680 SPACE SOMETHING WE'RE VERY 1006 00:40:51,680 --> 00:40:53,680 EXCITED ABOUT AS YOU PROBABLY 1007 00:40:53,680 --> 00:40:55,200 KNOW DELIVERY OF GENOME EDITORS 1008 00:40:55,200 --> 00:40:57,920 INTO THE NERVOUS SYSTEM IS A BIG 1009 00:40:57,920 --> 00:40:58,200 CHALLENGE. 1010 00:40:58,200 --> 00:41:00,320 SO ONE OF THE COMPONENTS OF THE 1011 00:41:00,320 --> 00:41:05,040 PROGRAM WE HAVE IS ACTUALLY A 1012 00:41:05,040 --> 00:41:06,440 PRIZE COMPETITION FOR NON-VIRAL 1013 00:41:06,440 --> 00:41:08,560 TECHNOLOGIES TO DELIVER GENOME 1014 00:41:08,560 --> 00:41:12,240 EDITORS ACROSS THE BLOOD-BRAIN 1015 00:41:12,240 --> 00:41:13,920 BARRIER INTO SUBSTANTIAL NUMBERS 1016 00:41:13,920 --> 00:41:15,840 OF CLINICAL RELEVANT CELLS IN 1017 00:41:15,840 --> 00:41:16,280 THE BRAIN. 1018 00:41:16,280 --> 00:41:18,280 THIS IS A PRIZE COMPETITION, IN 1019 00:41:18,280 --> 00:41:21,560 THAT REGARD QUITE DIFFERENT THAN 1020 00:41:21,560 --> 00:41:23,520 A TYPICAL NIH GRANT. 1021 00:41:23,520 --> 00:41:24,640 IT'S OPEN TO ANYONE. 1022 00:41:24,640 --> 00:41:26,400 AND I'LL PUT THE LINK IN THE 1023 00:41:26,400 --> 00:41:27,880 CHAT AS WE'RE TALKING SO PEOPLE 1024 00:41:27,880 --> 00:41:31,280 CAN SEE IT AND WE WOULD VERY 1025 00:41:31,280 --> 00:41:35,560 MUCH EN ENCOURAGE PEOPLE TO 1026 00:41:35,560 --> 00:41:36,440 DISSEMINATE THE INFORMATION. 1027 00:41:36,440 --> 00:41:38,520 IF WE CAN DEVELOP A NON-VIRAL 1028 00:41:38,520 --> 00:41:39,800 TECHNOLOGY TO CROSS THE 1029 00:41:39,800 --> 00:41:41,560 BLOOD-BRAIN BARRIER THAT WOULD 1030 00:41:41,560 --> 00:41:45,560 HAVE A HUGE IMPACT ON THE FIELD 1031 00:41:45,560 --> 00:41:46,840 AND TO TREAT MULTIPLE GENETIC 1032 00:41:46,840 --> 00:41:48,600 DISEASES THAT AFFECT THE NERVOUS 1033 00:41:48,600 --> 00:41:48,960 SYSTEM. 1034 00:41:48,960 --> 00:41:51,560 SO I THINK I'LL STOP THERE AND 1035 00:41:51,560 --> 00:41:56,040 HAND IT OVER I BELIEVE TO GOPA. 1036 00:41:56,040 --> 00:42:00,320 >> THANK YOU VERY MUCH, P.J. 1037 00:42:00,320 --> 00:42:01,480 WE CAN SEE YOU NOW. 1038 00:42:01,480 --> 00:42:02,480 >> I GUESS THAT'S GOOD. 1039 00:42:02,480 --> 00:42:03,920 I DON'T KNOW. 1040 00:42:03,920 --> 00:42:09,640 PROS AND CONS. 1041 00:42:09,640 --> 00:42:10,880 >> DR. GOPA RAYCHAUDHURI OF THE 1042 00:42:10,880 --> 00:42:13,520 FDA IS NEXT. 1043 00:42:13,520 --> 00:42:14,680 >> THANKS VERY MUCH. 1044 00:42:14,680 --> 00:42:16,440 I'D LIKE TO THANK MEETING 1045 00:42:16,440 --> 00:42:18,200 ORGANIZERS FOR THE OPPORTUNITY 1046 00:42:18,200 --> 00:42:19,280 TO PARTICIPATE IN THIS 1047 00:42:19,280 --> 00:42:21,920 CONFERENCE, TO BE PART OF THE 1048 00:42:21,920 --> 00:42:22,360 PANEL. 1049 00:42:22,360 --> 00:42:24,560 THE TOPIC PARTNERSHIPS AND 1050 00:42:24,560 --> 00:42:25,600 COLLABORATION IS INCREDIBLY 1051 00:42:25,600 --> 00:42:26,960 IMPORTANT, REALLY TIMELY GIVEN 1052 00:42:26,960 --> 00:42:27,840 THE TREMENDOUS EXPANSION WE'RE 1053 00:42:27,840 --> 00:42:31,120 SEEING IN THE GENE THERAPY FIELD 1054 00:42:31,120 --> 00:42:31,560 RIGHT NOW. 1055 00:42:31,560 --> 00:42:34,840 SO I WANT TO START BY SAYING 1056 00:42:34,840 --> 00:42:39,280 THAT THE FDA'S HIGHLY COMMITTED 1057 00:42:39,280 --> 00:42:41,400 TO FACILITATE THE DEVELOPMENT OF 1058 00:42:41,400 --> 00:42:43,800 SAFE AND EFFECTIVE THERAPIES TO 1059 00:42:43,800 --> 00:42:45,240 ADDRESS UNMET MEDICAL NEEDS FOR 1060 00:42:45,240 --> 00:42:47,120 PATIENTS WITH RARE AND VERY RARE 1061 00:42:47,120 --> 00:42:47,560 DISEASES. 1062 00:42:47,560 --> 00:42:50,680 THIS IS A VERY HIGH PRIORITY FOR 1063 00:42:50,680 --> 00:42:55,000 US, AND WE'RE TAKING A 1064 00:42:55,000 --> 00:42:55,640 MULTI-PRONGED APPROACH. 1065 00:42:55,640 --> 00:43:00,680 SO I WANT TO SHARE A FEW AREAS 1066 00:43:00,680 --> 00:43:01,520 WHERE WE'RE ENGAGED AND HAPPY 1067 00:43:01,520 --> 00:43:02,640 OVER THE COURSE OF THE SESSION 1068 00:43:02,640 --> 00:43:04,400 TO EXPAND ON ANY OF THESE. 1069 00:43:04,400 --> 00:43:09,480 ONE AREA IS PROVIDING FDA 1070 00:43:09,480 --> 00:43:09,720 GUIDANCE. 1071 00:43:09,720 --> 00:43:12,280 THERE IS A LOT OF -- THERE'S A 1072 00:43:12,280 --> 00:43:15,040 LOT OF NEED TO PROVIDE GUIDANCE 1073 00:43:15,040 --> 00:43:16,240 TO HELP FACILITATE THE 1074 00:43:16,240 --> 00:43:17,440 DEVELOPMENT OF THESE NOVEL 1075 00:43:17,440 --> 00:43:18,080 PRODUCTS. 1076 00:43:18,080 --> 00:43:18,960 AND SO OUR OFFICE OF 1077 00:43:18,960 --> 00:43:20,520 THERAPEUTICS PRODUCTS HAS BEEN 1078 00:43:20,520 --> 00:43:22,560 VERY PROLIFIC IN PROVIDING 1079 00:43:22,560 --> 00:43:24,120 GUIDANCE FOR GENE THERAPIES AS 1080 00:43:24,120 --> 00:43:26,840 WELL AS FOR TOPICS THAT ARE 1081 00:43:26,840 --> 00:43:29,520 PARTICULARLY IMPORTANT FOR RARE 1082 00:43:29,520 --> 00:43:29,800 DISEASES. 1083 00:43:29,800 --> 00:43:33,320 THAT'S ONE AREA. 1084 00:43:33,320 --> 00:43:34,520 WE ENCOURAGE SPONSORS TO 1085 00:43:34,520 --> 00:43:36,680 COMMUNICATE WITH US THROUGHOUT 1086 00:43:36,680 --> 00:43:37,800 THE ENTIRE PRODUCT DEVELOPMENT 1087 00:43:37,800 --> 00:43:38,800 PROCESS. 1088 00:43:38,800 --> 00:43:40,640 BUT FOR RARE DISEASES IT'S 1089 00:43:40,640 --> 00:43:43,280 PARTICULARLY IMPORTANT TO ENGAGE 1090 00:43:43,280 --> 00:43:45,560 EARLY, EVEN IN THE PRE-IND 1091 00:43:45,560 --> 00:43:45,760 SPACE. 1092 00:43:45,760 --> 00:43:47,640 I CAN TOUCH ON SOME OF THE 1093 00:43:47,640 --> 00:43:50,600 PROGRAMS THAT WE HAVE THAT ARE 1094 00:43:50,600 --> 00:43:52,040 ENCOURAGING PRODUCT DEVELOPERS 1095 00:43:52,040 --> 00:43:54,240 TO COME IN EARLY TO DISCUSS THE 1096 00:43:54,240 --> 00:43:55,200 DEVELOPMENT PLANS BEFORE YOU 1097 00:43:55,200 --> 00:43:59,040 EVEN GET STARTED. 1098 00:43:59,040 --> 00:44:01,800 I'M SURE YOU'RE AWARE WE HAVE A 1099 00:44:01,800 --> 00:44:04,400 NUMBER OF DIFFERENT PROGRAMS, 1100 00:44:04,400 --> 00:44:05,400 EXPEDITED PROGRAMS, TO 1101 00:44:05,400 --> 00:44:08,240 FACILITATE PRODUCT DEVELOPMENT 1102 00:44:08,240 --> 00:44:10,760 FOR DISEASES THAT ARE SERIOUS OR 1103 00:44:10,760 --> 00:44:11,640 LIFE-THREATENING, AND CERTAINLY 1104 00:44:11,640 --> 00:44:14,360 MANY, MANY RARE DISEASES FALL 1105 00:44:14,360 --> 00:44:15,880 INTO THAT CATEGORY. 1106 00:44:15,880 --> 00:44:19,760 AND THEN THE FOURTH THING I WANT 1107 00:44:19,760 --> 00:44:21,080 TO EMPHASIZE IS PARTNERSHIPS, 1108 00:44:21,080 --> 00:44:22,520 THE TOPIC OF WHAT WE'RE 1109 00:44:22,520 --> 00:44:23,720 DISCUSSING TODAY. 1110 00:44:23,720 --> 00:44:27,640 WE VERY ACTIVELY ENGAGED IN 1111 00:44:27,640 --> 00:44:28,080 PARTNERSHIPS. 1112 00:44:28,080 --> 00:44:30,800 WE'RE VERY -- WE'RE AN INTEGRAL 1113 00:44:30,800 --> 00:44:35,640 PART OF THE BESPOKE CONSORTIUM, 1114 00:44:35,640 --> 00:44:38,040 BUT MANY EARS BECAUSE THIS IS 1115 00:44:38,040 --> 00:44:41,560 CRITICAL TO HAVE A SEAT AT THE 1116 00:44:41,560 --> 00:44:42,720 TABLE AND WORK WITH COMMUNITIES 1117 00:44:42,720 --> 00:44:45,240 AS THESE PRODUCTS ARE DEVELOPED. 1118 00:44:45,240 --> 00:44:47,640 WHAT I WANT TO REALLY EMPHASIZE 1119 00:44:47,640 --> 00:44:49,760 IS THE ROLE OF PATIENT 1120 00:44:49,760 --> 00:44:52,840 ENGAGEMENT IN THIS. 1121 00:44:52,840 --> 00:44:54,840 IT'S CRITICAL FOR PATIENTS TO 1122 00:44:54,840 --> 00:44:57,240 WORK RIGHT FROM DAY 1 WITH 1123 00:44:57,240 --> 00:44:58,560 PRODUCT DEVELOPERS TO CONVEY 1124 00:44:58,560 --> 00:45:02,000 WHAT IT IS THAT IS IMPORTANT TO 1125 00:45:02,000 --> 00:45:02,640 THE PATIENT GROUP, IN TERMS OF 1126 00:45:02,640 --> 00:45:05,760 WHAT IS IT YOU'RE LOOKING FOR IN 1127 00:45:05,760 --> 00:45:06,360 A THERAPY THAT WOULD IMPACT 1128 00:45:06,360 --> 00:45:09,960 QUALITY OF LIFE. 1129 00:45:09,960 --> 00:45:12,800 WE WOULD LOVE TO FIND CURES BUT 1130 00:45:12,800 --> 00:45:15,640 THAT MAY NOT BE POSSIBLE BUT 1131 00:45:15,640 --> 00:45:16,280 THERAPIES COULD PROVIDE 1132 00:45:16,280 --> 00:45:16,960 SIGNIFICANT BENEFIT. 1133 00:45:16,960 --> 00:45:18,160 IT'S IMPORTANT TO HEAR THE 1134 00:45:18,160 --> 00:45:20,120 PATIENT VOICE IN THAT RIGHT FROM 1135 00:45:20,120 --> 00:45:21,200 THE VERY BEGINNING. 1136 00:45:21,200 --> 00:45:25,040 AND SO AT THE FDA, WE WANT VERY 1137 00:45:25,040 --> 00:45:27,240 MUCH TO HEAR WHAT THE PATIENT 1138 00:45:27,240 --> 00:45:29,400 PERSPECTIVE IS, AND WE HAVE 1139 00:45:29,400 --> 00:45:30,120 PATIENT LISTENING SESSIONS 1140 00:45:30,120 --> 00:45:32,480 DESIGNED SPECIFICALLY FOR THAT 1141 00:45:32,480 --> 00:45:32,840 PURPOSE. 1142 00:45:32,840 --> 00:45:34,960 WE'RE IN LISTENING MODE TO HEAR 1143 00:45:34,960 --> 00:45:36,960 FROM PATIENTS, FROM CAREGIVERS, 1144 00:45:36,960 --> 00:45:38,040 ABOUT WHAT YOUR EXPERIENCE IS, 1145 00:45:38,040 --> 00:45:40,280 WHAT IS IT THAT WOULD MAKE AN 1146 00:45:40,280 --> 00:45:42,840 IMPACT, BECAUSE ALL OF THAT 1147 00:45:42,840 --> 00:45:44,520 FACTORS INTO OUR THINKING AS WE 1148 00:45:44,520 --> 00:45:46,360 LOOK AT THE PRODUCTS THAT ARE 1149 00:45:46,360 --> 00:45:49,880 BEING DEVELOPED AND THE 1150 00:45:49,880 --> 00:45:51,400 INDICATIONS FOR THOSE PRODUCTS. 1151 00:45:51,400 --> 00:45:56,320 I WANT TO EMPHASIZE THE PATIENT 1152 00:45:56,320 --> 00:45:57,200 ENGAGEMENT IS CRITICALLY 1153 00:45:57,200 --> 00:45:58,440 IMPORTANT, IMPORTANT FROM DAY 1 1154 00:45:58,440 --> 00:46:01,280 AS PRODUCT DEVELOPMENT PLANS ARE 1155 00:46:01,280 --> 00:46:02,920 BEING FORMULATED. 1156 00:46:02,920 --> 00:46:04,720 AND WE'RE OF COURSE -- WE TRY AS 1157 00:46:04,720 --> 00:46:07,400 MUCH AS WE CAN TO HAVE WORKSHOPS 1158 00:46:07,400 --> 00:46:09,840 AND TOWN HALL MEETINGS, ET 1159 00:46:09,840 --> 00:46:11,600 CETERA, TO SHARE OUR CURRENT 1160 00:46:11,600 --> 00:46:12,760 THINKING ON DIFFERENT ISSUES AS 1161 00:46:12,760 --> 00:46:14,880 THEY COME UP. 1162 00:46:14,880 --> 00:46:16,480 THIS IS AN INCREDIBLY 1163 00:46:16,480 --> 00:46:17,800 FAST-MOVING FIELD. 1164 00:46:17,800 --> 00:46:19,880 I'VE NOT BEEN INVOLVED, YOU 1165 00:46:19,880 --> 00:46:21,240 KNOW, IN RESEARCH THAT HAS BEEN 1166 00:46:21,240 --> 00:46:23,360 MOVING AS FAST AS THE FIELD OF 1167 00:46:23,360 --> 00:46:25,240 GENE THERAPIES HAS BEEN MOVING. 1168 00:46:25,240 --> 00:46:27,080 SO WE'RE LEARNING ALONG THE WAY 1169 00:46:27,080 --> 00:46:27,280 TOO. 1170 00:46:27,280 --> 00:46:28,960 WE'RE LEARNING FROM THE 1171 00:46:28,960 --> 00:46:31,720 RESEARCHERS AND WE'RE LEARNING 1172 00:46:31,720 --> 00:46:34,480 FROM THE OUTCOMES OF TRIALS, AND 1173 00:46:34,480 --> 00:46:36,320 WE'RE EVOLVING IN OUR THINKING. 1174 00:46:36,320 --> 00:46:38,920 THAT'S WHY PUBLIC COMMUNICATION 1175 00:46:38,920 --> 00:46:40,320 THROUGH WORKSHOPS, TOWN HALLS, 1176 00:46:40,320 --> 00:46:42,040 IS SO CRITICAL. 1177 00:46:42,040 --> 00:46:45,880 AND WE'RE DOING THAT AS WELL IN 1178 00:46:45,880 --> 00:46:49,400 OUR PARTNERSHIP AND ROLE IN THE 1179 00:46:49,400 --> 00:46:49,800 BGTC. 1180 00:46:49,800 --> 00:46:52,120 I JUST WANT TO TOUCH ON ONE 1181 00:46:52,120 --> 00:46:54,280 THING IN PARTICULAR BECAUSE YOU 1182 00:46:54,280 --> 00:46:59,240 MAY HAVE HEARD THIS FROM PETER 1183 00:46:59,240 --> 00:47:00,320 MARX, OUR CENTER DIRECTOR, 1184 00:47:00,320 --> 00:47:03,720 THINKING ABOUT POTENTIAL OF 1185 00:47:03,720 --> 00:47:07,760 USING ACCELERATED APPROVAL 1186 00:47:07,760 --> 00:47:09,320 PATHWAY FOR RARE DISEASES AS A 1187 00:47:09,320 --> 00:47:11,360 MEANS TO GET APPROVALS AND 1188 00:47:11,360 --> 00:47:14,760 ACCESS FOR PATIENTS IN SOME OF 1189 00:47:14,760 --> 00:47:18,760 THESE THERAPIES, EARLIER IN THE 1190 00:47:18,760 --> 00:47:19,360 PROCESS. 1191 00:47:19,360 --> 00:47:20,160 ACCELERATED APPROVAL PATHWAY 1192 00:47:20,160 --> 00:47:22,560 DEPENDS ON DEMONSTRATION OF 1193 00:47:22,560 --> 00:47:23,280 SUBSTANTIAL EVIDENCE OF 1194 00:47:23,280 --> 00:47:25,760 EFFECTIVENESS, BASED ON THE 1195 00:47:25,760 --> 00:47:29,120 SURROGATE ENDPOINT OR AN 1196 00:47:29,120 --> 00:47:30,000 INTERMEDIATE CLINICAL ENDPOINT 1197 00:47:30,000 --> 00:47:34,280 THAT IS VERY LIKELY TO PREDICT 1198 00:47:34,280 --> 00:47:35,120 CLINICAL BENEFIT. 1199 00:47:35,120 --> 00:47:38,640 THIS IS SOMETHING THAT WE'RE 1200 00:47:38,640 --> 00:47:39,960 THINKING VERY CAREFULLY ABOUT, 1201 00:47:39,960 --> 00:47:41,920 AND IF THE CIRCUMSTANCES ARE 1202 00:47:41,920 --> 00:47:44,560 RIGHT, IF THE DATA ARE 1203 00:47:44,560 --> 00:47:47,360 SUPPORTIVE OF USING THIS PATHWAY 1204 00:47:47,360 --> 00:47:49,560 TO MOVE TOWARDS LICENSURE, THIS 1205 00:47:49,560 --> 00:47:51,560 IS SOMETHING THAT WE'RE 1206 00:47:51,560 --> 00:47:53,080 DISCUSSING AND DELIBERATING, BUT 1207 00:47:53,080 --> 00:47:55,280 IT'S SOMETHING TO THINK ABOUT 1208 00:47:55,280 --> 00:47:57,920 BECAUSE AS YOU THINK ABOUT 1209 00:47:57,920 --> 00:48:01,000 CLINICAL DEVELOPMENT PATHWAY 1210 00:48:01,000 --> 00:48:02,280 THAT YOU'RE GOING TO BE TAKING 1211 00:48:02,280 --> 00:48:03,360 IT'S IMPORTANT TO THINK RIGHT 1212 00:48:03,360 --> 00:48:05,560 FROM THE BEGINNING ABOUT WHAT 1213 00:48:05,560 --> 00:48:06,320 REGULATORY PATHWAY YOU'RE GOING 1214 00:48:06,320 --> 00:48:10,680 TO BE USING TO MOVE TOWARDS 1215 00:48:10,680 --> 00:48:17,720 LICENSURE, AND ENSURING SOLID 1216 00:48:17,720 --> 00:48:20,000 DATA TO DEMONSTRATE THIS IS AN 1217 00:48:20,000 --> 00:48:22,520 APPROPRIATE SURROGATE ENDPOINT 1218 00:48:22,520 --> 00:48:24,600 OR APPROPRIATE CLINICAL ENDPOINT 1219 00:48:24,600 --> 00:48:25,800 IS CRITICALLY IMPORTANT. 1220 00:48:25,800 --> 00:48:27,320 NATURAL HISTORY STUDIES ARE 1221 00:48:27,320 --> 00:48:28,320 INCREDIBLY IMPORTANT. 1222 00:48:28,320 --> 00:48:31,600 SO THE THINKING ABOUT -- I LIKE 1223 00:48:31,600 --> 00:48:34,240 TO SAY THAT -- THINK ABOUT WHAT 1224 00:48:34,240 --> 00:48:36,640 THE END GAME IS YOU WANT AND 1225 00:48:36,640 --> 00:48:40,040 WORK BACKWARDS AS YOU WORK ON 1226 00:48:40,040 --> 00:48:44,480 DEVELOPMENT OF YOUR CLINICAL 1227 00:48:44,480 --> 00:48:44,880 PATHWAYS. 1228 00:48:44,880 --> 00:48:48,720 AND THEN I JUST WANT TO END BY 1229 00:48:48,720 --> 00:48:52,160 SAYING WE'RE TALKING IN THE 1230 00:48:52,160 --> 00:48:54,040 BGTC, FOCUS ON DISEASES THAT 1231 00:48:54,040 --> 00:48:56,200 CURRENTLY HAVE NO COMMERCIAL 1232 00:48:56,200 --> 00:48:56,480 INTEREST. 1233 00:48:56,480 --> 00:48:59,520 BUT THE ENTIRE GOAL OF THIS 1234 00:48:59,520 --> 00:49:01,240 ACTIVITY AND THIS PROCESS AND 1235 00:49:01,240 --> 00:49:02,800 THIS PROGRAM IS TO REALLY GET IT 1236 00:49:02,800 --> 00:49:06,160 TO THE POINT WHERE DISEASES THAT 1237 00:49:06,160 --> 00:49:07,040 CURRENTLY DON'T HAVE COMMERCIAL 1238 00:49:07,040 --> 00:49:10,000 INTEREST GET TO THE POINT WHERE 1239 00:49:10,000 --> 00:49:10,960 THERE IS COMMERCIAL INTEREST. 1240 00:49:10,960 --> 00:49:15,360 THIS IS REALLY GOING TO REQUIRE 1241 00:49:15,360 --> 00:49:18,240 INNOVATION IN MANUFACTURING, 1242 00:49:18,240 --> 00:49:19,520 INNOVATION IN TECHNOLOGY, AND 1243 00:49:19,520 --> 00:49:20,720 REGULATORS HAVE A CRITICAL ROLE 1244 00:49:20,720 --> 00:49:20,960 HERE. 1245 00:49:20,960 --> 00:49:24,560 WE HAVE TO LOOK AT STREAMLINING 1246 00:49:24,560 --> 00:49:26,160 REGULATORY PATHWAYS, STILL BASED 1247 00:49:26,160 --> 00:49:29,040 ON SCIENCE, TO ENSURE SAFETY AND 1248 00:49:29,040 --> 00:49:29,320 EFFICACY. 1249 00:49:29,320 --> 00:49:32,320 BUT WE HAVE TO STREAMLINE THE 1250 00:49:32,320 --> 00:49:35,720 REGULATORY PATHWAY IN ORDER TO 1251 00:49:35,720 --> 00:49:37,000 HAVE PRODUCT DEVELOPERS SEE A 1252 00:49:37,000 --> 00:49:40,840 MEANS TO GET TO THAT ENDPOINT OF 1253 00:49:40,840 --> 00:49:41,640 LICENSURE FOR THERAPEUTICS 1254 00:49:41,640 --> 00:49:44,120 PRODUCTS FOR THESE VERY RARE 1255 00:49:44,120 --> 00:49:44,440 DISEASES. 1256 00:49:44,440 --> 00:49:46,960 THAT'S SOMETHING THAT WE ARE 1257 00:49:46,960 --> 00:49:49,920 LITERALLY WORKING ON EVERY DAY, 1258 00:49:49,920 --> 00:49:52,080 AND DECISIONS THAT WE MAKE ARE 1259 00:49:52,080 --> 00:49:55,720 BASED ON DATA, AND THAT'S WHY 1260 00:49:55,720 --> 00:49:59,800 THINGS LIKE THE BGTC AND OTHER 1261 00:49:59,800 --> 00:50:00,560 CONSORTIA AND CLINICAL TRIALS 1262 00:50:00,560 --> 00:50:02,160 ONGOING ARE SO CRITICALLY 1263 00:50:02,160 --> 00:50:03,400 IMPORTANT BECAUSE WE CAN LEARN 1264 00:50:03,400 --> 00:50:06,760 FROM THAT AS TO WHERE IS IT 1265 00:50:06,760 --> 00:50:07,800 APPROPRIATE TO STREAMLINE. 1266 00:50:07,800 --> 00:50:09,120 YOU KNOW, BECAUSE IT HAS TO BE 1267 00:50:09,120 --> 00:50:09,640 BASED ON DATA. 1268 00:50:09,640 --> 00:50:11,720 BUT THAT IS THE ULTIMATE GOAL, 1269 00:50:11,720 --> 00:50:13,480 THAT IS THE ONLY WAY WE'RE GOING 1270 00:50:13,480 --> 00:50:15,880 TO BE ABLE TO GET 1271 00:50:15,880 --> 00:50:16,840 COMMERCIALIZATION OF THESE 1272 00:50:16,840 --> 00:50:19,480 THERAPIES FOR THESE VERY RARE 1273 00:50:19,480 --> 00:50:20,920 DISEASES. 1274 00:50:20,920 --> 00:50:24,760 I WILL END AND MAYBE WE'LL TALK 1275 00:50:24,760 --> 00:50:28,120 ABOUT THIS LATER, BUT ESPECIALLY 1276 00:50:28,120 --> 00:50:29,800 IN THE RARE DISEASE SPACE IT'S 1277 00:50:29,800 --> 00:50:30,800 CRITICALLY IMPORTANT TO LOOK AT 1278 00:50:30,800 --> 00:50:32,200 PRODUCT DEVELOPMENT FROM A 1279 00:50:32,200 --> 00:50:33,520 GLOBAL PERSPECTIVE BECAUSE THERE 1280 00:50:33,520 --> 00:50:35,840 MAY NOT BE ENOUGH PATIENTS IN 1281 00:50:35,840 --> 00:50:38,360 ANY ONE COUNTRY. 1282 00:50:38,360 --> 00:50:41,400 TERRY WAS SAYING HIS SON IS THE 1283 00:50:41,400 --> 00:50:43,600 ONLY PERSON IN ALL OF CANADA, 1284 00:50:43,600 --> 00:50:46,800 THERE MAY BE ONLY 20 OR 30 IN 1285 00:50:46,800 --> 00:50:49,040 THE U.S., AND COMPANIES MAY NOT 1286 00:50:49,040 --> 00:50:50,720 WANT TO JUMP IN AND DEVELOP A 1287 00:50:50,720 --> 00:50:50,960 THERAPY. 1288 00:50:50,960 --> 00:50:53,240 IF YOU LOOK AT THE GLOBAL 1289 00:50:53,240 --> 00:50:55,320 POPULATION, HOWEVER, MAYBE WE 1290 00:50:55,320 --> 00:50:57,480 GET THAT CRITICAL NUMBER THAT 1291 00:50:57,480 --> 00:50:59,280 WILL HAVE, YOU KNOW, THE 1292 00:50:59,280 --> 00:51:00,360 MANUFACTURERS AND COMPANIES SAY 1293 00:51:00,360 --> 00:51:02,120 YES, WE WANT TO INVEST IN THIS 1294 00:51:02,120 --> 00:51:03,120 FOR DEVELOPMENT. 1295 00:51:03,120 --> 00:51:05,720 MAYBE WE CAN TALK MORE ABOUT THE 1296 00:51:05,720 --> 00:51:06,600 REGULATORY COMPONENT OF THAT 1297 00:51:06,600 --> 00:51:10,320 DOWN THE ROAD BUT I THINK THAT 1298 00:51:10,320 --> 00:51:12,160 PRODUCT MANUFACTURERS NEED TO 1299 00:51:12,160 --> 00:51:14,040 LOOK BROADER THAN JUST HERE OR 1300 00:51:14,040 --> 00:51:15,480 THERE BUT LOOK GLOBALLY FOR 1301 00:51:15,480 --> 00:51:18,000 THESE PRODUCT DEVELOPMENT 1302 00:51:18,000 --> 00:51:19,000 EFFORTS. 1303 00:51:19,000 --> 00:51:21,400 >> THANK YOU, GOPA. 1304 00:51:21,400 --> 00:51:24,440 I'LL SEGUE TO SAY WE'LL HAVE 1305 00:51:24,440 --> 00:51:26,520 PLENTY OF TIME FOR DISCUSSION SO 1306 00:51:26,520 --> 00:51:29,160 GET YOUR QUESTIONS READY AS YOU 1307 00:51:29,160 --> 00:51:33,200 LISTEN TO OUR CLEANUP HITTER, 1308 00:51:33,200 --> 00:51:33,680 DR. TRAVIS LEWIS. 1309 00:51:33,680 --> 00:51:34,640 >> THANK YOU. 1310 00:51:34,640 --> 00:51:35,680 THANKS FOR INVITING ME. 1311 00:51:35,680 --> 00:51:37,720 I WAS INVITED TO BE THE 1312 00:51:37,720 --> 00:51:39,000 CHEERLEADER FOR THIS GROUP, AS 1313 00:51:39,000 --> 00:51:42,720 WE TALKED ABOUT. 1314 00:51:42,720 --> 00:51:47,920 BUT IT'S AN EASY JOB TO HAVE 1315 00:51:47,920 --> 00:51:48,120 HERE. 1316 00:51:48,120 --> 00:51:50,080 I'M A MOVEMENTS DISORDER 1317 00:51:50,080 --> 00:51:51,720 NEUROLOGIST, Ph.D. WORK 1318 00:51:51,720 --> 00:51:55,040 STUDYING GENE THERAPY AS A 1319 00:51:55,040 --> 00:51:56,280 PLATFORM FOR NEURODEGENERATIVE 1320 00:51:56,280 --> 00:51:57,320 DISEASES WITH THIS ACT IDEA 1321 00:51:57,320 --> 00:52:04,000 THESE CAN BE A PLATFORM AND WAYS 1322 00:52:04,000 --> 00:52:06,600 TO STREAMLINE DEVELOPMENT I'M A 1323 00:52:06,600 --> 00:52:08,120 NEUROLOGIST WORKING IN INDUSTRY, 1324 00:52:08,120 --> 00:52:11,720 VARIOUS EXPERIENCES, WORKED IN 1325 00:52:11,720 --> 00:52:16,360 SMALL BIOTECH, CURRENTLY WORK 1326 00:52:16,360 --> 00:52:19,520 FOR PREVAIL THERAPEUTICS AND CAN 1327 00:52:19,520 --> 00:52:21,000 COMMENT ON HOW WE STARTED WITH 1328 00:52:21,000 --> 00:52:22,440 THE SAME ISSUES. 1329 00:52:22,440 --> 00:52:25,400 IT'S A DIFFICULT AND CONSTANTLY 1330 00:52:25,400 --> 00:52:25,840 DEVELOPING FIELD. 1331 00:52:25,840 --> 00:52:27,720 WITH A LOT OF DEDICATED PEOPLE. 1332 00:52:27,720 --> 00:52:35,160 ABOUT 2 1/2 YEARS AGO ACQUIRED 1333 00:52:35,160 --> 00:52:37,880 BY ELI LILLY, NOW A BIGGER 1334 00:52:37,880 --> 00:52:39,720 PHARMA, IT'S INSPIRING TO SEE 1335 00:52:39,720 --> 00:52:41,520 HOW THESE CONTINUE DEVELOPMENT 1336 00:52:41,520 --> 00:52:44,000 AND ADVANCEMENT OF THESE 1337 00:52:44,000 --> 00:52:45,000 THERAPEUTICS CAN BE PUSHED 1338 00:52:45,000 --> 00:52:48,080 FORWARD IN A BIG PHARMA-TYPE 1339 00:52:48,080 --> 00:52:52,960 SPACE, ALSO IN SMALL BIOTECH 1340 00:52:52,960 --> 00:52:53,200 SPACE. 1341 00:52:53,200 --> 00:52:55,960 I CAN SPEAK TO HOW RESOURCES AT 1342 00:52:55,960 --> 00:52:58,760 THE NIH, FDA, NINDS, HAVE BEEN 1343 00:52:58,760 --> 00:53:01,840 WORKING ON IT, EXTREMELY 1344 00:53:01,840 --> 00:53:04,240 IMPORTANT ACROSS REALLY EVERY 1345 00:53:04,240 --> 00:53:07,920 FUNCTION AS WE DEVELOP THESE 1346 00:53:07,920 --> 00:53:09,200 THERAPIES. 1347 00:53:09,200 --> 00:53:14,920 AT PREVAIL WE STUDY RARE 1348 00:53:14,920 --> 00:53:20,320 GENETICS FORMS, FRONTOTEMPORAL, 1349 00:53:20,320 --> 00:53:21,760 PARKINSON'S WITH MUTATION AND 1350 00:53:21,760 --> 00:53:25,080 GAUCHER'S DISEASE, A RARE AND 1351 00:53:25,080 --> 00:53:35,560 RAPIDLY PROGRESSING FORM OF 1352 00:53:39,400 --> 00:53:40,040 GAUCHER'S. 1353 00:53:40,040 --> 00:53:42,480 IT'S INSPIRING TO SEE, AND THESE 1354 00:53:42,480 --> 00:53:43,880 RESOURCES WOULD BE HELPFUL TO US 1355 00:53:43,880 --> 00:53:47,720 TO BE ABLE TO USE GOING FORWARD, 1356 00:53:47,720 --> 00:53:49,040 FOR NEW COMPANIES JOINING THAT 1357 00:53:49,040 --> 00:53:50,240 DON'T HAVE AS MUCH EXPERIENCE IN 1358 00:53:50,240 --> 00:53:55,480 THE FIELD SO THEY DON'T START 1359 00:53:55,480 --> 00:53:57,680 FROM ZERO BUT CAN START FROM A 1360 00:53:57,680 --> 00:53:58,560 RUNNING START. 1361 00:53:58,560 --> 00:54:01,080 IT'S REALLY INSPIRING AND 1362 00:54:01,080 --> 00:54:02,600 IMPORTANT AND SHOWS COMMITMENT 1363 00:54:02,600 --> 00:54:04,800 THESE ORGANIZATIONS HAVE TOWARDS 1364 00:54:04,800 --> 00:54:08,080 GENE THERAPY. 1365 00:54:08,080 --> 00:54:09,200 THERE'S REAL POTENTIAL HERE, IF 1366 00:54:09,200 --> 00:54:10,840 NOT TO CURE TO CHANGE THE 1367 00:54:10,840 --> 00:54:12,160 TRAJECTORY OF THE DISEASES. 1368 00:54:12,160 --> 00:54:16,960 I'LL ALSO COMMENT ON HOW 1369 00:54:16,960 --> 00:54:18,640 IMPORTANT INTERACTION WITH THE 1370 00:54:18,640 --> 00:54:20,160 PATIENT ADVOCACY ORGANIZATIONS 1371 00:54:20,160 --> 00:54:23,240 ARE FOR US. 1372 00:54:23,240 --> 00:54:25,640 IT'S JUST A LOT OF HARD WORK, 1373 00:54:25,640 --> 00:54:27,760 AND HAVING THESE INSPIRING 1374 00:54:27,760 --> 00:54:28,960 MOMENTS, INTERACTIONS WITH THE 1375 00:54:28,960 --> 00:54:31,040 FAMILIES AND PATIENTS THAT ARE 1376 00:54:31,040 --> 00:54:34,120 LIVING WITH THE DISEASES, THAT 1377 00:54:34,120 --> 00:54:35,640 WE HAVE THE OPPORTUNITY TO 1378 00:54:35,640 --> 00:54:36,960 SUPPORT IS REALLY MEANINGFUL. 1379 00:54:36,960 --> 00:54:39,160 I THINK AS WE WERE TALKING ABOUT 1380 00:54:39,160 --> 00:54:41,040 EARLIER TODAY, EACH INDICATION 1381 00:54:41,040 --> 00:54:45,840 HAS ITS OWN CHALLENGES. 1382 00:54:45,840 --> 00:54:48,080 AND DIFFERENT CHALLENGES, RIGHT? 1383 00:54:48,080 --> 00:54:49,920 DIFFERENT RATES OF PROGRESSION, 1384 00:54:49,920 --> 00:54:51,880 DIFFERENT POPULATIONS, DIFFERENT 1385 00:54:51,880 --> 00:54:54,320 NUMBERS OF PATIENTS. 1386 00:54:54,320 --> 00:54:57,720 SO EACH INDICATION IS UNIQUE. 1387 00:54:57,720 --> 00:54:59,560 SO THAT EARLY INTERACTION WITH 1388 00:54:59,560 --> 00:55:00,760 PATIENTS, KNOWING WHAT THEY 1389 00:55:00,760 --> 00:55:02,080 NEED, KNOWING WHAT WE'RE TRYING 1390 00:55:02,080 --> 00:55:05,040 TO DO FOR THEM, HOW WE CAN WORK 1391 00:55:05,040 --> 00:55:06,000 TOGETHER IS EXTREMELY IMPORTANT. 1392 00:55:06,000 --> 00:55:09,520 AND I CAN SPEAK FOR MYSELF, FOR 1393 00:55:09,520 --> 00:55:11,400 THE COMPANIES I WORKED FOR, THAT 1394 00:55:11,400 --> 00:55:13,480 THE PEOPLE IN INDUSTRY ARE 1395 00:55:13,480 --> 00:55:15,240 PASSIONATE ABOUT DOING THIS. 1396 00:55:15,240 --> 00:55:16,720 THESE ARE CHALLENGING. 1397 00:55:16,720 --> 00:55:19,440 THESE ARE SMALL DISEASES. 1398 00:55:19,440 --> 00:55:22,240 WE'RE DOING THIS BECAUSE WE WANT 1399 00:55:22,240 --> 00:55:23,280 TO SHOW GENE THERAPIES CAN 1400 00:55:23,280 --> 00:55:25,680 CHANGE THE COURSE OF THESE 1401 00:55:25,680 --> 00:55:26,280 DISEASES. 1402 00:55:26,280 --> 00:55:30,440 THESE ARE THE OPPORTUNITIES TO 1403 00:55:30,440 --> 00:55:30,680 DO THAT. 1404 00:55:30,680 --> 00:55:33,920 >> THANK YOU SO MUCH, DR. LEWIS. 1405 00:55:33,920 --> 00:55:38,120 WHAT A GREAT SEGUE INTO THE 1406 00:55:38,120 --> 00:55:38,520 DISCUSSION. 1407 00:55:38,520 --> 00:55:41,360 I'M JUST THRILLED, I KNEW I 1408 00:55:41,360 --> 00:55:45,400 WOULD BE, BECAUSE WE HAD A PREP 1409 00:55:45,400 --> 00:55:45,800 CALL. 1410 00:55:45,800 --> 00:55:46,520 WONDERFUL PANELISTS KNOW SO MUCH 1411 00:55:46,520 --> 00:55:48,480 ABOUT THE THINGS WE WANT TO KNOW 1412 00:55:48,480 --> 00:55:48,720 ABOUT. 1413 00:55:48,720 --> 00:55:51,560 WE WANT TO KNOW ESPECIALLY HOW 1414 00:55:51,560 --> 00:55:53,960 WE CAN HELP ADVANCE GENE 1415 00:55:53,960 --> 00:55:58,280 THERAPIES IN OUR VARIOUS 1416 00:55:58,280 --> 00:56:04,160 DISEASES. 1417 00:56:04,160 --> 00:56:05,560 TO OPEN THE DISCUSSION, GIVE YOU 1418 00:56:05,560 --> 00:56:08,120 MORE TIME TO THINK ABOUT YOUR 1419 00:56:08,120 --> 00:56:09,320 QUESTIONS, I PREPARED TWO 1420 00:56:09,320 --> 00:56:10,720 QUESTIONS OF MY OWN. 1421 00:56:10,720 --> 00:56:12,560 IF YOU HAVE A BURNING ONE, 1422 00:56:12,560 --> 00:56:14,120 INTERRUPT ME AFTER THE FIRST 1423 00:56:14,120 --> 00:56:14,720 ONE. 1424 00:56:14,720 --> 00:56:18,040 WE'LL GO TO THAT. 1425 00:56:18,040 --> 00:56:21,240 THE FIRST QUESTION TO KICK THIS 1426 00:56:21,240 --> 00:56:25,720 DISCUSSION OFF SEVERAL OF YOU 1427 00:56:25,720 --> 00:56:27,920 MENTIONED THIS IS DIFFICULT. 1428 00:56:27,920 --> 00:56:30,960 I BET EVERYBODY IN THIS ROOM, 1429 00:56:30,960 --> 00:56:34,480 YOU KNOW, KNOWS THAT GENE 1430 00:56:34,480 --> 00:56:37,120 THERAPY HAS BEEN ON OUR PLATE 1431 00:56:37,120 --> 00:56:40,520 FOR CLOSE TO 30 YEARS. 1432 00:56:40,520 --> 00:56:43,040 THE FIRST EXPERIMENTS WERE IN 1433 00:56:43,040 --> 00:56:44,360 THE 1990s. 1434 00:56:44,360 --> 00:56:47,080 AT LAST COUNT THE FDA HAS 1435 00:56:47,080 --> 00:56:51,440 APPROVED I THINK WE'RE UP TO 27 1436 00:56:51,440 --> 00:56:52,880 GENE THERAPIES. 1437 00:56:52,880 --> 00:56:57,800 MOST OF THOSE IN CANCER OR BLOOD 1438 00:56:57,800 --> 00:56:58,280 DISEASES. 1439 00:56:58,280 --> 00:57:02,840 ONLY ONE THAT I KNOW OF, MAYBE 1440 00:57:02,840 --> 00:57:07,440 TWO NOW, ARE IN NEUROLOGICAL 1441 00:57:07,440 --> 00:57:08,720 CONDITIONS. 1442 00:57:08,720 --> 00:57:14,320 SO I WILL QUOTE DR. PETER MARKS 1443 00:57:14,320 --> 00:57:17,080 THAT GOPA BROUGHT UP EARLIER 1444 00:57:17,080 --> 00:57:19,160 WHEN HE SPOKE AT ORPHAN DRUG 1445 00:57:19,160 --> 00:57:24,480 CONGRESS A COUPLE MONTHS AGO, HE 1446 00:57:24,480 --> 00:57:25,920 ITERATED WHAT WE WERE SO 1447 00:57:25,920 --> 00:57:27,120 HOPEFUL, GENE THERAPY IS GOING 1448 00:57:27,120 --> 00:57:29,080 TO BE THE CURE FOR ALL OF US, 1449 00:57:29,080 --> 00:57:29,840 LET'S GO. 1450 00:57:29,840 --> 00:57:36,000 IT'S PROVING TO BE MORE 1451 00:57:36,000 --> 00:57:36,440 DIFFICULT. 1452 00:57:36,440 --> 00:57:39,280 ISSUES ARE STILL PLAGUING 1453 00:57:39,280 --> 00:57:40,840 PROMISING GENE THERAPY PROGRAMS. 1454 00:57:40,840 --> 00:57:46,520 THERE WAS PETER, CBER DIRECTOR, 1455 00:57:46,520 --> 00:57:48,080 SAYING THIS IS PROVING TO BE 1456 00:57:48,080 --> 00:57:49,560 HARDER THAN WE HOPED. 1457 00:57:49,560 --> 00:57:53,520 WITH THAT IN MIND I'D LIKE TO 1458 00:57:53,520 --> 00:57:55,160 ASK THESE WONDERFUL PANELISTS, 1459 00:57:55,160 --> 00:57:57,800 FIRST OF ALL, LET ME ADD TOO IN 1460 00:57:57,800 --> 00:58:01,280 OUR DISEASE GROUP WE HAD ABOUT 1461 00:58:01,280 --> 00:58:03,960 14 DIFFERENT COMPANIES ADVANCING 1462 00:58:03,960 --> 00:58:06,160 GENE THERAPY AND GENE EDITING IN 1463 00:58:06,160 --> 00:58:09,240 OUR RARE DISEASE. 1464 00:58:09,240 --> 00:58:13,280 THAT NUMBER HAS DROPPED. 1465 00:58:13,280 --> 00:58:15,280 WE'VE HAD BIG COMPANIES ABANDON 1466 00:58:15,280 --> 00:58:16,520 THEIR GENE THERAPY PROGRAMS, NOT 1467 00:58:16,520 --> 00:58:18,880 JUST IN OUR DISEASE BUT IN OTHER 1468 00:58:18,880 --> 00:58:21,280 DISEASES AS WELL. 1469 00:58:21,280 --> 00:58:26,840 WE'VE HAD SMALLER COMPANIES 1470 00:58:26,840 --> 00:58:31,760 ABANDON -- WE'VE HAD OTHER 1471 00:58:31,760 --> 00:58:32,840 COMPANIES ABANDON, YOU KNOW, OUR 1472 00:58:32,840 --> 00:58:35,560 DISEASE AND ALL THEIR OTHER GENE 1473 00:58:35,560 --> 00:58:37,880 THERAPY PROGRAMS TO RETURN TO 1474 00:58:37,880 --> 00:58:42,280 SMALL MOLECULES AS A MORE VIABLE 1475 00:58:42,280 --> 00:58:43,680 FUTURE. 1476 00:58:43,680 --> 00:58:45,080 SO I'D LIKE TO ASK THE TOUGH 1477 00:58:45,080 --> 00:58:48,520 QUESTION OF THESE WONDERFUL 1478 00:58:48,520 --> 00:58:49,000 PANELISTS. 1479 00:58:49,000 --> 00:58:50,080 HOW HOPEFUL, HOW CONFIDENT ARE 1480 00:58:50,080 --> 00:58:51,680 YOU ABOUT THE FUTURE OF GENE 1481 00:58:51,680 --> 00:58:57,600 THERAPY FOR ALL OF OUR DISEASES 1482 00:58:57,600 --> 00:58:58,720 OR OTHER GENE-BASED THERAPEUTIC 1483 00:58:58,720 --> 00:59:00,880 APPROACHES? 1484 00:59:00,880 --> 00:59:07,440 1485 00:59:07,440 --> 00:59:11,000 >> I THINK THAT WE ARE ON THE 1486 00:59:11,000 --> 00:59:12,400 CUSP OF A CONVERGENCE. 1487 00:59:12,400 --> 00:59:14,600 I THINK THERE ARE A LOT OF 1488 00:59:14,600 --> 00:59:19,840 DIFFERENT AREAS, PUBLIC/PRIVATE 1489 00:59:19,840 --> 00:59:21,240 PARTNERSHIPS, INDIVIDUAL 1490 00:59:21,240 --> 00:59:22,600 DEVELOPMENT PIPELINES, RESEARCH 1491 00:59:22,600 --> 00:59:24,720 EFFORTS, THAT ARE EACH LOOKING 1492 00:59:24,720 --> 00:59:28,760 AT A SLIGHTLY DIFFERENT PIECE OF 1493 00:59:28,760 --> 00:59:29,680 THE PUZZLE. 1494 00:59:29,680 --> 00:59:33,520 SO LOOKING AT THE AAV VECTOR, 1495 00:59:33,520 --> 00:59:41,400 BGTC LOOKING AT THE PRE-CLINICAL 1496 00:59:41,400 --> 00:59:43,680 AND REGULATORY PLATFORM, AND I 1497 00:59:43,680 --> 00:59:46,240 THINK THAT THERE IS A TREMENDOUS 1498 00:59:46,240 --> 00:59:48,960 OPPORTUNITY FOR ALL OF THESE 1499 00:59:48,960 --> 00:59:51,480 EFFORTS TO CONVERGE AND 1500 00:59:51,480 --> 00:59:52,680 SYNERGIZE AND EACH ONE 1501 00:59:52,680 --> 00:59:54,720 ACCELERATING A LITTLE BIT OF THE 1502 00:59:54,720 --> 00:59:54,960 PATHWAY. 1503 00:59:54,960 --> 00:59:57,240 AND THEN YOU CAN BUILD MOMENTUM 1504 00:59:57,240 --> 00:59:59,920 FROM THE MANUFACTURING INTO THE 1505 00:59:59,920 --> 01:00:02,600 PRE-CLINICAL, INTO THE CLINICAL 1506 01:00:02,600 --> 01:00:02,800 TRIAL. 1507 01:00:02,800 --> 01:00:04,200 I'M PRETTY HOPEFUL. 1508 01:00:04,200 --> 01:00:07,640 I WAS JUST AT A CONFERENCE LAST 1509 01:00:07,640 --> 01:00:09,400 WEEK WITH AN ORGANIZATION CALLED 1510 01:00:09,400 --> 01:00:11,280 LADDERS TO CURES. 1511 01:00:11,280 --> 01:00:13,200 THERE HAS BEEN SUCH TREMENDOUS 1512 01:00:13,200 --> 01:00:18,840 WORK IN THE FIELD OF GENOME 1513 01:00:18,840 --> 01:00:20,640 SEQUENCING AND IDENTIFYING 1514 01:00:20,640 --> 01:00:22,160 INDIVIDUAL GENE ABERRATIONS FOR 1515 01:00:22,160 --> 01:00:25,320 THE DISEASES, ANDS WE'VE KIND OF 1516 01:00:25,320 --> 01:00:28,640 OF BEEN IN A TIME OF SPLITTING 1517 01:00:28,640 --> 01:00:30,280 THE NUANCE OF EACH INDIVIDUAL 1518 01:00:30,280 --> 01:00:33,000 GENE THAT HAS LED TO 7,000 AND 1519 01:00:33,000 --> 01:00:36,600 THEN 10,000 BECAUSE WE KEEP 1520 01:00:36,600 --> 01:00:37,440 FINDING SUB-SUB-SUBPOPULATIONS 1521 01:00:37,440 --> 01:00:38,000 OF THESE DISEASES. 1522 01:00:38,000 --> 01:00:40,760 WHAT THIS GROUP IS TRYING TO DO 1523 01:00:40,760 --> 01:00:43,040 IS LUMPING RATHER THAN SPLITTING 1524 01:00:43,040 --> 01:00:44,920 TO LOOK FOR COMMONALITIES, TO 1525 01:00:44,920 --> 01:00:46,440 LOOK FOR SHARED PATHWAYS, TO 1526 01:00:46,440 --> 01:00:48,720 LOOK FOR EFFORTS THAT CAN CREATE 1527 01:00:48,720 --> 01:00:50,280 MULTIPLE DISEASES WITH A SINGLE 1528 01:00:50,280 --> 01:00:52,560 TYPE OF THERAPY OR SINGLE DRUG 1529 01:00:52,560 --> 01:00:54,440 AND SO FORTH. 1530 01:00:54,440 --> 01:00:56,400 I THINK THE COMBINATION OF THIS 1531 01:00:56,400 --> 01:00:58,040 WORK IS REALLY PUTTING US RIGHT 1532 01:00:58,040 --> 01:01:00,680 ON THE CUSP OF WHAT I HOPE WILL 1533 01:01:00,680 --> 01:01:02,520 BE A SNOWBALL EFFORT WHERE WE 1534 01:01:02,520 --> 01:01:04,160 HAVE TWO OR THREE GENE THERAPIES 1535 01:01:04,160 --> 01:01:06,480 FOR RARE DISEASES TODAY, THAT WE 1536 01:01:06,480 --> 01:01:10,800 HAVE 20 IN FIVE YEARS AND 200 IN 1537 01:01:10,800 --> 01:01:12,600 A DECADE AND I THINK IT JUST 1538 01:01:12,600 --> 01:01:13,280 TAKES OFF. 1539 01:01:13,280 --> 01:01:13,600 >> WONDERFUL. 1540 01:01:13,600 --> 01:01:15,800 WHO ELSE WOULD LIKE TO 1541 01:01:15,800 --> 01:01:16,560 CONTRIBUTE TO THAT? 1542 01:01:16,560 --> 01:01:18,640 >> YEAH, I WOULD ECHO THAT. 1543 01:01:18,640 --> 01:01:20,960 I THINK IT'S AN EXCITING TIME. 1544 01:01:20,960 --> 01:01:24,320 I THINK IN THE LAST 10, 20 YEARS 1545 01:01:24,320 --> 01:01:26,760 WE'VE LEARNED ALL OF THE 1546 01:01:26,760 --> 01:01:27,040 CHALLENGES. 1547 01:01:27,040 --> 01:01:30,080 WE'RE STILL LEARNING MANY 1548 01:01:30,080 --> 01:01:30,480 CHALLENGES. 1549 01:01:30,480 --> 01:01:34,760 I THINK THAT THESE PLATFORM 1550 01:01:34,760 --> 01:01:35,720 INITIATIVES AND REGULATORY 1551 01:01:35,720 --> 01:01:36,320 INITIATIVES STREAMLINING THE 1552 01:01:36,320 --> 01:01:37,320 DEVELOPMENT ARE GOING TO GO A 1553 01:01:37,320 --> 01:01:39,040 LONG WAY. 1554 01:01:39,040 --> 01:01:41,120 THESE ARE THE HUGE HURDLES WE'VE 1555 01:01:41,120 --> 01:01:43,320 RUN INTO THAT TAKE A LOT OF 1556 01:01:43,320 --> 01:01:43,560 WORK. 1557 01:01:43,560 --> 01:01:45,720 YOU HAVE THE RIGHT DRUG, RIGHT 1558 01:01:45,720 --> 01:01:47,160 VECTOR, BUT HOW DO YOU MAKE 1559 01:01:47,160 --> 01:01:51,280 ENOUGH, HOW DO YOU MAKE IT IN A 1560 01:01:51,280 --> 01:01:52,800 WAY THAT YOU CAN USE YOUR 1561 01:01:52,800 --> 01:01:55,440 CLINICAL TRIAL AND PHASE 3 AND 1562 01:01:55,440 --> 01:01:55,960 APPROVAL. 1563 01:01:55,960 --> 01:01:57,440 THESE ARE -- A LOT STARTED OUT 1564 01:01:57,440 --> 01:01:58,840 IN RESEARCH LABS, SO BRINGING 1565 01:01:58,840 --> 01:02:02,680 THEM TO THE CLINIC IS A HUGE 1566 01:02:02,680 --> 01:02:03,240 LEAP. 1567 01:02:03,240 --> 01:02:06,640 AND I THINK THE SUPPORT FOR 1568 01:02:06,640 --> 01:02:08,280 SMALLER UPSTART BIOTECHS TO HAVE 1569 01:02:08,280 --> 01:02:12,000 THE RESOURCES ON HOW TO DO THIS 1570 01:02:12,000 --> 01:02:13,520 CORRECTLY, FROM DAY 1, I THINK 1571 01:02:13,520 --> 01:02:15,480 WILL STREAMLINE THE NUMBER OF 1572 01:02:15,480 --> 01:02:18,240 PROGRAMS OUT THERE. 1573 01:02:18,240 --> 01:02:21,760 AND I AGREE, WE'RE LEARNING ALL 1574 01:02:21,760 --> 01:02:25,160 THE OF THE HURDLES, WE'RE 1575 01:02:25,160 --> 01:02:26,800 LEARNING THE DIFFERENT 1576 01:02:26,800 --> 01:02:30,160 ADMINISTRATION AND CHALLENGES 1577 01:02:30,160 --> 01:02:33,800 BUT I THINK THAT WE'RE COMING TO 1578 01:02:33,800 --> 01:02:36,440 THAT NEXUS OF KNOWLEDGE AND 1579 01:02:36,440 --> 01:02:38,280 THERE'S A REALLY EXCITING 1580 01:02:38,280 --> 01:02:39,880 OPPORTUNITY TO MOVE FORWARD. 1581 01:02:39,880 --> 01:02:42,560 >> AND GOPA, COULD YOU TELL US A 1582 01:02:42,560 --> 01:02:44,720 LITTLE BIT ABOUT -- WE'RE 1583 01:02:44,720 --> 01:02:47,160 APPRECIATIVE OF THE FACT THAT 1584 01:02:47,160 --> 01:02:49,440 THE FDA AND CBER ARE PREPARING 1585 01:02:49,440 --> 01:02:52,320 FOR SUCCESS AND GROWING THEIR 1586 01:02:52,320 --> 01:02:52,920 RESOURCES. 1587 01:02:52,920 --> 01:02:56,240 I WOULD LIKE TO THANK ADVOCATES 1588 01:02:56,240 --> 01:02:59,080 IN THE ROOM WHO HELPED CONVINCE 1589 01:02:59,080 --> 01:03:01,040 CONGRESS THAT CBER NEEDED THOSE 1590 01:03:01,040 --> 01:03:02,400 ADDITIONAL RESOURCES TO ADVANCE 1591 01:03:02,400 --> 01:03:05,240 THIS FIELD. 1592 01:03:05,240 --> 01:03:07,200 COULD YOU TELL US ABOUT THAT 1593 01:03:07,200 --> 01:03:08,720 PREPARATION, THE GROWTH OF WHAT 1594 01:03:08,720 --> 01:03:11,320 USED TO BE THE OFFICE OF TISSUES 1595 01:03:11,320 --> 01:03:13,080 AND ADVANCED THERAPIES, INTO THE 1596 01:03:13,080 --> 01:03:18,160 SUPER OFFICE OF THE OFFICE OF 1597 01:03:18,160 --> 01:03:19,040 THERAPEUTIC PRODUCTS. 1598 01:03:19,040 --> 01:03:20,000 >> THANKS, RON. 1599 01:03:20,000 --> 01:03:23,080 I CANNOT THANK THE ADVOCACY 1600 01:03:23,080 --> 01:03:26,080 GROUPS ENOUGH BECAUSE OUR 1601 01:03:26,080 --> 01:03:27,600 REVIEWERS ARE AN INCREDIBLY 1602 01:03:27,600 --> 01:03:29,360 DEDICATED GROUP OF PEOPLE. 1603 01:03:29,360 --> 01:03:31,760 BUT IF YOU SEE THE NUMBER OF 1604 01:03:31,760 --> 01:03:32,760 APPLICATIONS THAT HAVE COME IN, 1605 01:03:32,760 --> 01:03:34,480 AND THIS IS JUST A REFLECTION OF 1606 01:03:34,480 --> 01:03:37,440 THE ADVANCEMENTS IN THE FIELD, 1607 01:03:37,440 --> 01:03:40,640 IT'S SIGH ROCKETED. 1608 01:03:40,640 --> 01:03:43,040 WE NEEDED THE RESOURCES 1609 01:03:43,040 --> 01:03:43,800 ABSOLUTELY. 1610 01:03:43,800 --> 01:03:47,080 SO, OFFICE OF TISSUES AND 1611 01:03:47,080 --> 01:03:49,520 ADVANCED THERAPIES HAS NOW BEEN 1612 01:03:49,520 --> 01:03:51,120 INCREASED INTO THE STATUS OF A 1613 01:03:51,120 --> 01:03:53,760 SUPER OFFICE, WHICH MEANS THAT 1614 01:03:53,760 --> 01:03:56,040 IT GETS MORE RESOURCES, THERE 1615 01:03:56,040 --> 01:03:59,360 ARE PLANS TO HIRE OVER 100 NEW 1616 01:03:59,360 --> 01:04:03,720 REVIEW STAFF, WHICH WILL GO A 1617 01:04:03,720 --> 01:04:06,880 LONG WAY TOWARDS SPEEDING UP 1618 01:04:06,880 --> 01:04:08,600 THINGS IN TERMS OF 1619 01:04:08,600 --> 01:04:10,200 COMMUNICATIONS, FEEDBACK, ET 1620 01:04:10,200 --> 01:04:10,440 CETERA. 1621 01:04:10,440 --> 01:04:12,480 I KNOW THERE'S A LOT OF -- AND 1622 01:04:12,480 --> 01:04:14,680 WE GO OUT THERE AND WE SAY COME 1623 01:04:14,680 --> 01:04:16,200 TO US, YOU KNOW, EARLY AND COME 1624 01:04:16,200 --> 01:04:18,520 TO US OFTEN BUT WE ALSO KNOW 1625 01:04:18,520 --> 01:04:23,440 THAT A NUMBER OF REQUESTS FOR 1626 01:04:23,440 --> 01:04:25,320 MEETINGS, WE CAN'T GIVE ALL OF 1627 01:04:25,320 --> 01:04:27,040 THE MEETINGS THAT ARE REQUESTED. 1628 01:04:27,040 --> 01:04:30,160 EVEN IF WE WANT TO. 1629 01:04:30,160 --> 01:04:31,440 BUT THE INCREASE IN RESOURCES, 1630 01:04:31,440 --> 01:04:33,760 YOU KNOW, WILL REALLY HELP A 1631 01:04:33,760 --> 01:04:33,960 LOT. 1632 01:04:33,960 --> 01:04:36,240 I DO WANT TO COMMENT ON THE 1633 01:04:36,240 --> 01:04:37,800 QUESTION THAT YOU ASKED ABOUT, 1634 01:04:37,800 --> 01:04:41,520 YOU KNOW, DO YOU FEEL OPTIMISTIC 1635 01:04:41,520 --> 01:04:42,600 ABOUT THE FUTURE. 1636 01:04:42,600 --> 01:04:43,920 I ABSOLUTELY DO. 1637 01:04:43,920 --> 01:04:47,640 THE REASON I DO IS BECAUSE WHAT 1638 01:04:47,640 --> 01:04:52,120 WE'RE SEEING HERE AROUND IN THE 1639 01:04:52,120 --> 01:04:54,200 BGTC AND OTHER ORGANIZATIONS IS 1640 01:04:54,200 --> 01:04:56,160 THE COLLABORATIVE EFFORT THAT 1641 01:04:56,160 --> 01:04:58,040 PEOPLE ARE PUTTING INTO DEVELOP 1642 01:04:58,040 --> 01:04:58,560 THESE PRODUCTS. 1643 01:04:58,560 --> 01:05:01,760 INDUSTRY CAN'T DO IT ALONE. 1644 01:05:01,760 --> 01:05:02,400 ACADEMIC RESEARCH INSTITUTIONS 1645 01:05:02,400 --> 01:05:03,520 CAN'T DO IT ALONE. 1646 01:05:03,520 --> 01:05:06,360 FDA CAN'T DO IT ALONE OF THE 1647 01:05:06,360 --> 01:05:07,440 PATIENT ORGANIZATIONS HAVE A 1648 01:05:07,440 --> 01:05:08,200 TREMENDOUS IMPACT. 1649 01:05:08,200 --> 01:05:11,600 BUT YOU CAN'T DO IT ALONE. 1650 01:05:11,600 --> 01:05:14,120 WHEN WE COME TOGETHER, AND WORK 1651 01:05:14,120 --> 01:05:16,440 TOGETHER, THAT'S WHERE THE 1652 01:05:16,440 --> 01:05:17,440 EFFICIENCIES COME IN. 1653 01:05:17,440 --> 01:05:19,400 AND THE REASON I FEEL OPTIMISTIC 1654 01:05:19,400 --> 01:05:22,480 IS BECAUSE IT MAY FEEL LIKE IT'S 1655 01:05:22,480 --> 01:05:25,200 MOVING SLOWLY, AND WE FEEL LIKE 1656 01:05:25,200 --> 01:05:28,720 IT'S MOVING SLOWLY, BUT WE ALSO 1657 01:05:28,720 --> 01:05:30,560 FEEL THAT IN ORDER -- YOU KNOW, 1658 01:05:30,560 --> 01:05:32,760 WE NEED TO DO RIGHT BY THE 1659 01:05:32,760 --> 01:05:33,120 PATIENT. 1660 01:05:33,120 --> 01:05:34,680 IF WE'RE TO APPROVE ANY 1661 01:05:34,680 --> 01:05:36,720 PRODUCTS, THERE HAS TO BE 1662 01:05:36,720 --> 01:05:39,640 DEMONSTRATION OF SAFETY AND 1663 01:05:39,640 --> 01:05:40,000 EFFICACY. 1664 01:05:40,000 --> 01:05:42,200 AND SO EFFICIENCY DOESN'T MEAN 1665 01:05:42,200 --> 01:05:43,280 CUTTING CORNERS. 1666 01:05:43,280 --> 01:05:45,480 IT SIMPLY MEANS HOW EFFECTIVELY 1667 01:05:45,480 --> 01:05:47,560 DO WE GET TO THAT ENDPOINT. 1668 01:05:47,560 --> 01:05:48,880 AND THERE ARE EFFICIENCIES THAT 1669 01:05:48,880 --> 01:05:51,600 CAN BE BUILT IN THROUGH 1670 01:05:51,600 --> 01:05:52,400 COLLABORATION, THROUGH 1671 01:05:52,400 --> 01:05:53,000 STREAMLINING, AND THE 1672 01:05:53,000 --> 01:05:55,680 STREAMLINING IS GOING TO BE 1673 01:05:55,680 --> 01:05:57,080 BASED ON DATA. 1674 01:05:57,080 --> 01:05:59,360 SO WHAT I REALLY ENCOURAGE 1675 01:05:59,360 --> 01:06:02,120 PATIENT GROUPS TO DO IS TO 1676 01:06:02,120 --> 01:06:03,440 PARTICIPATE IN NATURAL HISTORY 1677 01:06:03,440 --> 01:06:04,520 STUDY. 1678 01:06:04,520 --> 01:06:08,240 THAT DATA IS ABSOLUTELY REALLY 1679 01:06:08,240 --> 01:06:08,680 CRITICAL. 1680 01:06:08,680 --> 01:06:10,000 AND ADVOCATE TO HAVE YOUR VOICE 1681 01:06:10,000 --> 01:06:11,160 HEARD IN TERMS OF YOUR 1682 01:06:11,160 --> 01:06:13,600 VIEWPOINTS ON WHAT IS IMPORTANT 1683 01:06:13,600 --> 01:06:15,320 TO THE PATIENT COMMUNITY, WITH 1684 01:06:15,320 --> 01:06:17,520 THE PRODUCT DEVELOPERS, SO THAT 1685 01:06:17,520 --> 01:06:19,640 THEY UNDERSTAND WHAT IT IS 1686 01:06:19,640 --> 01:06:20,720 YOU'RE LOOKING FOR IN THE 1687 01:06:20,720 --> 01:06:22,360 PRODUCTS THAT THEY ARE 1688 01:06:22,360 --> 01:06:22,960 DEVELOPING. 1689 01:06:22,960 --> 01:06:24,840 WE FIND THESE PATIENT 1690 01:06:24,840 --> 01:06:27,680 ENGAGEMENTS TO BE INCREDIBLY 1691 01:06:27,680 --> 01:06:28,960 HELPFUL IN UNDERSTANDING WHAT IS 1692 01:06:28,960 --> 01:06:31,280 IMPORTANT IN TERMS OF THE FINAL 1693 01:06:31,280 --> 01:06:32,960 INDICATION, AND I THINK PRODUCT 1694 01:06:32,960 --> 01:06:34,200 DEVELOPERS WOULD BENEFIT FROM 1695 01:06:34,200 --> 01:06:37,120 THAT ALSO. 1696 01:06:37,120 --> 01:06:39,960 >> RON, COULD I ANSWER THAT? 1697 01:06:39,960 --> 01:06:42,160 >> GO, P.J. 1698 01:06:42,160 --> 01:06:45,800 >> YEAH, I'M VERY OPTIMISTIC 1699 01:06:45,800 --> 01:06:50,520 BECAUSE, IN PART, THE BASIC 1700 01:06:50,520 --> 01:06:52,000 BIOLOGY, IF YOU'RE TALKING ABOUT 1701 01:06:52,000 --> 01:06:53,920 MONOGENIC DISEASE WE KNOW WHAT 1702 01:06:53,920 --> 01:06:55,320 THE PROBLEM IS AND WHAT A 1703 01:06:55,320 --> 01:06:57,400 SOLUTION IS, TO GET A WORKING 1704 01:06:57,400 --> 01:06:58,480 COPY OF THAT MUTANT GENE INTO 1705 01:06:58,480 --> 01:07:00,560 THE RIGHT CELLS AT THE RIGHT 1706 01:07:00,560 --> 01:07:01,440 TIME OF DEVELOPMENT. 1707 01:07:01,440 --> 01:07:02,920 IF WE CAN DO THAT, SOMETHING 1708 01:07:02,920 --> 01:07:05,840 GOOD IS GOING TO HAPPEN. 1709 01:07:05,840 --> 01:07:08,520 THAT IS TRUE OF ALL MONOGENIC 1710 01:07:08,520 --> 01:07:08,920 DISEASES. 1711 01:07:08,920 --> 01:07:11,520 THE PROBLEM IS GETTING IT TO 1712 01:07:11,520 --> 01:07:13,360 THOSE CELLS THAT ENOUGH IN THE 1713 01:07:13,360 --> 01:07:14,520 RIGHT TIME OF DEVELOPMENT, TO 1714 01:07:14,520 --> 01:07:17,960 THE FIRST POINT I THINK WHAT 1715 01:07:17,960 --> 01:07:19,040 WE'RE SEEING, THE NERVOUS 1716 01:07:19,040 --> 01:07:22,440 SYSTEM, IT COMES DOWN TO 1717 01:07:22,440 --> 01:07:24,320 DELIVERY AGAIN AS IT ALWAYS 1718 01:07:24,320 --> 01:07:26,800 DOES, HOW DO YOU GET TO ENOUGH 1719 01:07:26,800 --> 01:07:30,080 CELLS IN THE BRAIN, THE HUMAN 1720 01:07:30,080 --> 01:07:31,440 BRAIN IN PARTICULAR, PARTLY WHAT 1721 01:07:31,440 --> 01:07:33,840 MOTIVATED US TO DO THE CHALLENGE 1722 01:07:33,840 --> 01:07:36,240 THAT WE'RE DOING FORT NON-VIRAL 1723 01:07:36,240 --> 01:07:38,720 APPROACH TO DELIVER EFFICIENTLY 1724 01:07:38,720 --> 01:07:39,680 GENOME EDITORS ACROSS THE 1725 01:07:39,680 --> 01:07:40,840 BLOOD-BRAIN BARRIER. 1726 01:07:40,840 --> 01:07:42,480 THE RIGHT TIME OF DEVELOPMENT OF 1727 01:07:42,480 --> 01:07:44,720 DISEASE IS A TRICKY ONE, TO BE 1728 01:07:44,720 --> 01:07:46,160 HONEST, BECAUSE RIGHT NOW WE'RE 1729 01:07:46,160 --> 01:07:48,200 OFTEN TRYING TO DEVELOP DISEASES 1730 01:07:48,200 --> 01:07:50,440 AND PARTICULARLY WHEN LOOKING AT 1731 01:07:50,440 --> 01:07:51,560 NEURODEGENERATIVE DISEASES FOR 1732 01:07:51,560 --> 01:07:53,760 PATIENTS THAT HAVE EXPERIENCED 1733 01:07:53,760 --> 01:07:54,600 SOME DEGENERATION. 1734 01:07:54,600 --> 01:07:55,560 AND IT WOULD WORK BETTER OF 1735 01:07:55,560 --> 01:07:58,160 COURSE IF YOU GIVE THEM THE DRUG 1736 01:07:58,160 --> 01:08:01,720 BEFORE THAT PROCESS STARTS. 1737 01:08:01,720 --> 01:08:03,160 WE'VE SEEN THAT WITH 1738 01:08:03,160 --> 01:08:04,120 (INDISCERNIBLE) BUT THAT RAISES 1739 01:08:04,120 --> 01:08:06,080 A COMPLICATED OF HOW DO WE FIND 1740 01:08:06,080 --> 01:08:07,840 PATIENTS BEFORE THEY ARE 1741 01:08:07,840 --> 01:08:08,520 SYMPTOMATIC? 1742 01:08:08,520 --> 01:08:11,360 AND THAT GETS INTO A LOT OF 1743 01:08:11,360 --> 01:08:13,240 ISSUES ABOUT NEWBORN SCREENING 1744 01:08:13,240 --> 01:08:16,520 WHICH I'LL NOTE BUT NOT DIVE 1745 01:08:16,520 --> 01:08:16,720 INTO. 1746 01:08:16,720 --> 01:08:18,600 IF WE WANT TO SEE THE FULL 1747 01:08:18,600 --> 01:08:21,120 BENEFIT AT SOME POINT WE'LL HAVE 1748 01:08:21,120 --> 01:08:22,760 TO THINK ABOUT THAT. 1749 01:08:22,760 --> 01:08:29,000 SO THOSE ARE SOME OF THE MAIN 1750 01:08:29,000 --> 01:08:30,440 THINGS. 1751 01:08:30,440 --> 01:08:34,800 THE IDEA OF THERAPEUTIC 1752 01:08:34,800 --> 01:08:35,840 PLATFORM, LANGUAGE IN APPROVED 1753 01:08:35,840 --> 01:08:37,920 LEGISLATION STARTS TALKING ABOUT 1754 01:08:37,920 --> 01:08:41,440 DIRECTING FDA TO START THINKING 1755 01:08:41,440 --> 01:08:42,040 ABOUT THERAPEUTIC PLATFORMS, 1756 01:08:42,040 --> 01:08:44,600 PERSONALLY I THINK A VERY BIG 1757 01:08:44,600 --> 01:08:46,760 STEP FORWARD BECAUSE GENE 1758 01:08:46,760 --> 01:08:48,520 THERAPY AND GENE EDITING, IF 1759 01:08:48,520 --> 01:08:50,920 THOSE ARE NOT THERAPEUTIC 1760 01:08:50,920 --> 01:08:53,680 PLATFORMS, I DON'T WHAT THE WORD 1761 01:08:53,680 --> 01:08:54,760 THERAPEUTIC PLATFORM MEANS. 1762 01:08:54,760 --> 01:08:58,600 THIS IDEA OF USING THOSE 1763 01:08:58,600 --> 01:08:59,960 THERAPEUTIC PLATFORMS TO DEVELOP 1764 01:08:59,960 --> 01:09:01,560 ONE TREATMENT FOR ONE DISEASE AS 1765 01:09:01,560 --> 01:09:03,120 A TIME DOESN'T MAKE SENSE. 1766 01:09:03,120 --> 01:09:04,960 IT'S A SQUARE PEG IN A ROUND 1767 01:09:04,960 --> 01:09:05,480 HOLE SITUATION. 1768 01:09:05,480 --> 01:09:07,240 WE NEED TO ADDRESS THAT. 1769 01:09:07,240 --> 01:09:08,560 I'M HOPEFUL THAT WE'RE GOING TO 1770 01:09:08,560 --> 01:09:12,200 START SEEING SOME OF THAT KIND 1771 01:09:12,200 --> 01:09:13,400 OF CONVERGENCE. 1772 01:09:13,400 --> 01:09:14,840 BASED ON THOSE PERSPECTIVES I'M 1773 01:09:14,840 --> 01:09:18,760 QUITE OPTIMISTIC. 1774 01:09:18,760 --> 01:09:20,160 >> WONDERFUL INTERVENTIONS. 1775 01:09:20,160 --> 01:09:20,480 THANK YOU ALL. 1776 01:09:20,480 --> 01:09:22,800 I'D LIKE TO GO TO MY SECOND AND 1777 01:09:22,800 --> 01:09:27,080 LAST QUESTION BEFORE TURNING TO 1778 01:09:27,080 --> 01:09:29,680 EVERYBODY HERE AND ONLINE, THIS 1779 01:09:29,680 --> 01:09:31,440 IS DIRECTED TO THE FACT THAT 1780 01:09:31,440 --> 01:09:33,960 EVERYBODY ON THE PANEL HAS 1781 01:09:33,960 --> 01:09:35,920 REFERRED TO THE IMPORTANCE, 1782 01:09:35,920 --> 01:09:36,800 ESSENTIAL IMPORTANCE, OF 1783 01:09:36,800 --> 01:09:39,560 ENGAGING WITH PATIENTS AND THEIR 1784 01:09:39,560 --> 01:09:40,520 ADVOCACY ORGANIZATIONS IN 1785 01:09:40,520 --> 01:09:43,680 PREPARATION FOR GENE THERAPY AND 1786 01:09:43,680 --> 01:09:45,640 PARTICIPATION IN GENE THERAPY. 1787 01:09:45,640 --> 01:09:48,880 SO MY REQUEST IS THAT WE HONE IN 1788 01:09:48,880 --> 01:09:51,840 ON THAT. 1789 01:09:51,840 --> 01:09:56,320 WE HOME IN ON THAT ESSENTIAL 1790 01:09:56,320 --> 01:09:56,720 NATURE. 1791 01:09:56,720 --> 01:10:00,280 AND TRY TO TELL THESE WONDERFUL 1792 01:10:00,280 --> 01:10:02,160 ADVOCATES AND THEIR PATIENTS HOW 1793 01:10:02,160 --> 01:10:04,440 THIS -- YOU KNOW, TERRY 1794 01:10:04,440 --> 01:10:06,400 CONCLUDED HIS REMARKS AFTER 1795 01:10:06,400 --> 01:10:08,400 TELLING US WHAT ALL HE AND HIS 1796 01:10:08,400 --> 01:10:11,200 FAMILY TRIED TO DO ALONG THE WAY 1797 01:10:11,200 --> 01:10:14,920 TO PREPARE A SUCCESSFUL PATHWAY 1798 01:10:14,920 --> 01:10:16,880 FOR GENE THERAPY, CONCLUDED WITH 1799 01:10:16,880 --> 01:10:19,200 ALL THE THINGS THEY STILL NEED 1800 01:10:19,200 --> 01:10:20,040 TO DO. 1801 01:10:20,040 --> 01:10:22,360 AND HOW IMPORTANT THEY ARE TO 1802 01:10:22,360 --> 01:10:22,840 THE OVERALL PROJECT. 1803 01:10:22,840 --> 01:10:25,680 I'D LIKE TO ASK THE PANELISTS TO 1804 01:10:25,680 --> 01:10:27,640 HOME IN ON THE THINGS THAT 1805 01:10:27,640 --> 01:10:29,920 EVERYBODY IN THIS ROOM AND 1806 01:10:29,920 --> 01:10:33,680 ONLINE CAN DO NOW, EVEN THOUGH 1807 01:10:33,680 --> 01:10:42,840 THEY ARE NOT INVOLVED IN EITHER 1808 01:10:42,840 --> 01:10:44,400 PGT OR BGCT TO PREPARE THEIR 1809 01:10:44,400 --> 01:10:45,600 COMMUNITIES AND FAMILIES TO MOVE 1810 01:10:45,600 --> 01:10:47,000 SMARTLY ONCE THEY DO HAVE A GENE 1811 01:10:47,000 --> 01:10:55,480 THERAPY PROGRAM. 1812 01:10:55,480 --> 01:10:55,720 1813 01:10:55,720 --> 01:10:59,080 >> SO WHEN WE SET OUT IN 1814 01:10:59,080 --> 01:11:01,080 DECEMBER OF 2021 TO START THE 1815 01:11:01,080 --> 01:11:05,240 PROCESS OF IDENTIFYING THE 1816 01:11:05,240 --> 01:11:09,360 DISEASES THAT WOULD BE IN THE 1817 01:11:09,360 --> 01:11:11,240 BGCT PORTFOLIO WE TURNED TO 1818 01:11:11,240 --> 01:11:12,000 RESEARCH AND PATIENT COMMUNITIES 1819 01:11:12,000 --> 01:11:13,960 TO HELP US IN THAT PROCESS. 1820 01:11:13,960 --> 01:11:16,080 YOU WERE INSTRUMENTAL IN GETTING 1821 01:11:16,080 --> 01:11:20,000 US WHERE WE ARE TODAY. 1822 01:11:20,000 --> 01:11:22,840 THE PRIMARY GOAL WAS TO IDENTIFY 1823 01:11:22,840 --> 01:11:27,000 DISEASES WHERE THE BODY OF WORK 1824 01:11:27,000 --> 01:11:29,520 BOTH RESEARCH CLINICAL PATIENT 1825 01:11:29,520 --> 01:11:31,920 ADVOCACY HAD BROUGHT SOMETHING 1826 01:11:31,920 --> 01:11:33,000 TO AS CLOSE TO FIRST IN HUMAN 1827 01:11:33,000 --> 01:11:34,680 CLINICAL TRIALS AS POSSIBLE 1828 01:11:34,680 --> 01:11:38,160 BECAUSE WE WANTED TO USE THESE 1829 01:11:38,160 --> 01:11:39,920 AS TEST CASES TO DEMONSTRATE A 1830 01:11:39,920 --> 01:11:42,440 PROOF OF CONCEPT THAT YOU COULD 1831 01:11:42,440 --> 01:11:45,120 WITHIN A SET OF MULTIPLE 1832 01:11:45,120 --> 01:11:46,360 DISEASES THAT INVOLVED THE SAME 1833 01:11:46,360 --> 01:11:49,000 ROUTE OF ADMINISTRATION, THAT 1834 01:11:49,000 --> 01:11:53,480 YOU COULD STANDARDIZE THE 1835 01:11:53,480 --> 01:11:54,160 MANUFACTURING, PRE-CLINICAL 1836 01:11:54,160 --> 01:11:55,560 TESTING PROTOCOL, SO FORTH, SO 1837 01:11:55,560 --> 01:11:57,320 WE NEEDED THINGS CLOSE TO THE 1838 01:11:57,320 --> 01:11:58,840 START LINE, SO TO SPEAK. 1839 01:11:58,840 --> 01:12:00,240 WE PUT OUT A NOMINATION FORM 1840 01:12:00,240 --> 01:12:02,880 BECAUSE WE WANTED THIS TO BE A 1841 01:12:02,880 --> 01:12:08,240 LOW BAR OF ENTRY, AND WE HAD 62 1842 01:12:08,240 --> 01:12:10,000 DISEASES NOMINATED. 1843 01:12:10,000 --> 01:12:12,000 ALMOST ALL OF THOSE HAD AN 1844 01:12:12,000 --> 01:12:14,520 EXTREMELY HEAVY INFLUENCE FROM 1845 01:12:14,520 --> 01:12:16,720 THE PATIENT ADVOCACY COMMUNITY. 1846 01:12:16,720 --> 01:12:21,320 WE TAKELY WERE LOOKING FOR 1847 01:12:21,320 --> 01:12:30,960 OPPORTUNITIES THAT ALREADY HAD 1848 01:12:30,960 --> 01:12:32,840 CONTACT REGISTRIES, PATIENT 1849 01:12:32,840 --> 01:12:33,920 GROUPS, ENDPOINT IDENTIFIED, ALL 1850 01:12:33,920 --> 01:12:35,880 THINGS THE PATIENT COMMUNITIES 1851 01:12:35,880 --> 01:12:36,480 CAN CONTRIBUTE TO. 1852 01:12:36,480 --> 01:12:38,960 ALL EIGHT OF THE DISEASES THAT 1853 01:12:38,960 --> 01:12:41,840 ENDED UP IN THE PORTFOLIO HAVE A 1854 01:12:41,840 --> 01:12:44,240 CONTACT REGISTRY AND ONE OR MORE 1855 01:12:44,240 --> 01:12:44,720 ACTIVE PATIENT GROUPS. 1856 01:12:44,720 --> 01:12:46,200 THIS IS GOING TO BE REALLY 1857 01:12:46,200 --> 01:12:49,360 INSTRUMENTAL AS WE START TO MOVE 1858 01:12:49,360 --> 01:12:52,120 THROUGH THE MANUFACTURING INTO 1859 01:12:52,120 --> 01:12:53,480 CLINICAL TESTING, REGULATORY 1860 01:12:53,480 --> 01:12:55,040 SUBMISSIONS, SO FORTH, THAT 1861 01:12:55,040 --> 01:12:57,360 WE'RE IN THE PROCESS OF 1862 01:12:57,360 --> 01:12:58,560 REORIENTING THE STRUCTURE OF THE 1863 01:12:58,560 --> 01:13:01,400 CONSORTIUM A LITTLE BIT, FOR THE 1864 01:13:01,400 --> 01:13:03,680 FIRST 18 MONTHS LEADING UP TO 1865 01:13:03,680 --> 01:13:05,640 THE SELECTION OF THE CLINICAL 1866 01:13:05,640 --> 01:13:06,520 PORTFOLIO WERE FUNCTIONAL 1867 01:13:06,520 --> 01:13:09,160 ORGANIZED WITH A TEAM AROUND 1868 01:13:09,160 --> 01:13:10,680 MANUFACTURING CONSIDERATIONS. 1869 01:13:10,680 --> 01:13:14,400 WE HAD A TEAM AROUND 1870 01:13:14,400 --> 01:13:15,160 PRE-CLINICAL TESTING 1871 01:13:15,160 --> 01:13:17,200 CONSIDERATIONS, AROUND CLINICAL 1872 01:13:17,200 --> 01:13:18,120 CONSIDERATIONS, EACH HAD A 1873 01:13:18,120 --> 01:13:19,520 DEFINED ROLE IN THE PROCESS OF 1874 01:13:19,520 --> 01:13:20,200 DISEASE SELECTION. 1875 01:13:20,200 --> 01:13:21,520 NOW THAT WE HAVE EIGHT DISEASES 1876 01:13:21,520 --> 01:13:25,040 WE'RE IN THE PROCESS OF FORMING 1877 01:13:25,040 --> 01:13:29,560 CROSS-FUNCTIONAL TEAMS, HAVING A 1878 01:13:29,560 --> 01:13:30,840 REPRESENT FROM MANUFACTURER, 1879 01:13:30,840 --> 01:13:32,480 P.I. OF CLINICAL TRIAL, BRINGING 1880 01:13:32,480 --> 01:13:34,880 THE VOICES OF THE PATIENT IN SO 1881 01:13:34,880 --> 01:13:36,520 AS WE'RE SHINING A LIGHT, AS 1882 01:13:36,520 --> 01:13:39,160 WE'RE GOING TO THE FDA FOR 1883 01:13:39,160 --> 01:13:41,240 PRE-IND MEETINGS THE PATIENT IS 1884 01:13:41,240 --> 01:13:43,000 THERE AT EVERY STEP OF THE WAY 1885 01:13:43,000 --> 01:13:44,840 ENSURING THEIR VOICE IS HEARD 1886 01:13:44,840 --> 01:13:46,280 EARLY AND OFTEN THROUGHOUT OUR 1887 01:13:46,280 --> 01:13:47,280 PROCESS. 1888 01:13:47,280 --> 01:13:49,520 THAT'S THE KEY THING NOW FOR 1889 01:13:49,520 --> 01:13:51,200 PATIENTS TO CONTRIBUTE TO, 1890 01:13:51,200 --> 01:13:52,680 HELPING US GET TO THAT IND 1891 01:13:52,680 --> 01:13:56,240 SUBMISSION AND THAT CLINICAL 1892 01:13:56,240 --> 01:13:57,640 TRIAL. 1893 01:13:57,640 --> 01:13:59,400 >> MARVELOUS. 1894 01:13:59,400 --> 01:14:01,160 THANK YOU. 1895 01:14:01,160 --> 01:14:01,400 TRAVIS? 1896 01:14:01,400 --> 01:14:03,800 >> I'LL ECHO HOW IMPORTANT AND 1897 01:14:03,800 --> 01:14:05,320 WHY. 1898 01:14:05,320 --> 01:14:07,480 THOSE ARE TWO MAJOR CHALLENGES, 1899 01:14:07,480 --> 01:14:08,600 THE DRUG DEVELOPERS STRUGGLE 1900 01:14:08,600 --> 01:14:09,880 WITH THE MOST. 1901 01:14:09,880 --> 01:14:11,560 ESPECIALLY IN RARE DISEASES, 1902 01:14:11,560 --> 01:14:13,080 IDENTIFYING PATIENTS, KNOWING 1903 01:14:13,080 --> 01:14:15,480 WHAT THE BEST ENDPOINTS ARE THAT 1904 01:14:15,480 --> 01:14:17,600 ARE MEANINGFUL TO THOSE PATIENTS 1905 01:14:17,600 --> 01:14:19,960 AND IT'S RELATIVELY EASY TO FIND 1906 01:14:19,960 --> 01:14:21,680 THE SCIENTIFIC LEADERS IN THE 1907 01:14:21,680 --> 01:14:23,120 FIELD, CLINICAL LEADERS IN THE 1908 01:14:23,120 --> 01:14:24,360 FIELD, SPEAK TO THEM ABOUT WHAT 1909 01:14:24,360 --> 01:14:26,440 THEY KNOW ABOUT THE DISEASE. 1910 01:14:26,440 --> 01:14:28,960 BUT HEARING FROM THE PATIENT, 1911 01:14:28,960 --> 01:14:31,800 HEARING WHAT END POINTS MIGHT BE 1912 01:14:31,800 --> 01:14:36,600 MORE RELEVANT THAN MOTOR 1913 01:14:36,600 --> 01:14:39,440 ENDPOINTS OR SEIZURE FREQUENCY, 1914 01:14:39,440 --> 01:14:42,840 THINGS ALONG THOSE LINES, MAYBE 1915 01:14:42,840 --> 01:14:44,720 JUST BEING ABLE TO CHANGE THEM, 1916 01:14:44,720 --> 01:14:48,320 TO DO THINGS THAT ARE MORE 1917 01:14:48,320 --> 01:14:51,040 CLINICALLY MEANINGFUL FOR THE 1918 01:14:51,040 --> 01:14:55,040 PATIENTS IS SO IMPORTANT. 1919 01:14:55,040 --> 01:14:56,120 HAVING PATIENT REGISTRIES FOR 1920 01:14:56,120 --> 01:14:58,120 FAMILIES SO WHEN STUDIES COME 1921 01:14:58,120 --> 01:15:01,160 UP, YOU KNOW YOU'LL BE ABLE TO 1922 01:15:01,160 --> 01:15:03,200 IDENTIFY THOSE PATIENTS. 1923 01:15:03,200 --> 01:15:05,760 EVEN IF IT'S ONE OR TWO, LESS 1924 01:15:05,760 --> 01:15:16,240 THAN TEN PATIENTS A YEAR, THEY 1925 01:15:16,760 --> 01:15:17,160 ARE OUT THERE. 1926 01:15:17,160 --> 01:15:19,160 >> WHO ELSE WOULD LIKE TO -- 1927 01:15:19,160 --> 01:15:22,280 >> I WOULD ECHO POINTS ABOUT THE 1928 01:15:22,280 --> 01:15:24,480 NATURAL HISTORY STUDIES, 1929 01:15:24,480 --> 01:15:26,560 PARTICULARLY THE IMPORTANCE OF 1930 01:15:26,560 --> 01:15:28,280 HAVING CLINICAL OUTCOME MEASURES 1931 01:15:28,280 --> 01:15:29,840 FOR A CLINICAL TRIAL, BUT FOR 1932 01:15:29,840 --> 01:15:35,400 SOME OF THE GROUPS MAYBE COMING 1933 01:15:35,400 --> 01:15:37,160 INTO THIS, VERY KNEW, THE 1934 01:15:37,160 --> 01:15:40,040 IMPORTANCE OF HAVING AN ANIMAL 1935 01:15:40,040 --> 01:15:41,800 MODEL IS REALLY CRUCIAL AND I 1936 01:15:41,800 --> 01:15:43,760 THINK SOME FOLKS MAY NOT BE 1937 01:15:43,760 --> 01:15:45,960 AWARE THAT NOW THE DEVELOPING OF 1938 01:15:45,960 --> 01:15:47,600 AN ANIMAL MODEL, PARTICULARLY 1939 01:15:47,600 --> 01:15:49,640 MOUSE MODEL, IS A FAIRLY 1940 01:15:49,640 --> 01:15:50,640 STRAIGHTFORWARD THING TO DO. 1941 01:15:50,640 --> 01:15:54,240 YOU CAN CONTRACT WITH A COMPANY 1942 01:15:54,240 --> 01:15:55,360 FOR AROUND SOMETHING LIKE 1943 01:15:55,360 --> 01:15:58,520 $10,000 NOW, YOU CAN GET A MOUSE 1944 01:15:58,520 --> 01:16:00,080 MODEL MADE. 1945 01:16:00,080 --> 01:16:02,800 AND HERE GENOME EDITING IS JUST 1946 01:16:02,800 --> 01:16:04,800 A TECHNICAL TOOL, MAKES A BIG 1947 01:16:04,800 --> 01:16:06,520 DIFFERENCE, SO I THINK WHEN I 1948 01:16:06,520 --> 01:16:08,400 WAS RUNNING A RESEARCH LAB I 1949 01:16:08,400 --> 01:16:10,520 WORKED WITH SOME DISEASES WHERE 1950 01:16:10,520 --> 01:16:13,200 THERE'S ABOUT 100 PEOPLE 1951 01:16:13,200 --> 01:16:13,560 AFFECTED. 1952 01:16:13,560 --> 01:16:16,800 AND, YOU KNOW, FOR THEM RAISING 1953 01:16:16,800 --> 01:16:18,040 FUNDS WAS A CHALLENGE, BUT I 1954 01:16:18,040 --> 01:16:20,320 THINK RAISING FUNDS FOR A 1955 01:16:20,320 --> 01:16:21,560 SPECIFIC TARGET LIKE CAN WE 1956 01:16:21,560 --> 01:16:24,720 RAISE FUNDS TO GET A MOUSE MODEL 1957 01:16:24,720 --> 01:16:26,240 MADE, THAT'S SORT OF SPECIFIC 1958 01:16:26,240 --> 01:16:28,360 DELIVERABLE, IF WILL YOU, THAT 1959 01:16:28,360 --> 01:16:30,200 MIGHT BE A REAL BENEFIT FOR SOME 1960 01:16:30,200 --> 01:16:30,960 THESE DISEASES. 1961 01:16:30,960 --> 01:16:32,640 AGAIN, THE COST IS JUST NOT THAT 1962 01:16:32,640 --> 01:16:32,840 HIGH. 1963 01:16:32,840 --> 01:16:36,680 I WOULD ENFOURTH OF EN-- ENCOUE 1964 01:16:36,680 --> 01:16:38,960 WHO ARE VERY KNEW TO THINK ABOUT 1965 01:16:38,960 --> 01:16:39,160 THAT. 1966 01:16:39,160 --> 01:16:42,280 >> THANK YOU, P.J. 1967 01:16:42,280 --> 01:16:42,480 GOPA? 1968 01:16:42,480 --> 01:16:44,480 >> A QUICK COMMENT FOLLOWING UP 1969 01:16:44,480 --> 01:16:45,640 ON WHAT COURTNEY AND TRAVIS 1970 01:16:45,640 --> 01:16:49,800 SAID, WHICH I AGREE WITH 1971 01:16:49,800 --> 01:16:51,000 COMPLETELY, YOUR QUESTION WHAT 1972 01:16:51,000 --> 01:16:53,320 CAN PATIENT GROUPS DO NOW IN 1973 01:16:53,320 --> 01:16:55,000 PREPARATION. 1974 01:16:55,000 --> 01:16:57,680 WHAT IS REALLY CRITICALLY 1975 01:16:57,680 --> 01:16:59,440 IMPORTANT TO HAVE THE PATIENT 1976 01:16:59,440 --> 01:17:01,240 INPUT RIGHT THEY BEGINNING WHEN 1977 01:17:01,240 --> 01:17:02,560 PRODUCT DEVELOPERS ARE STARTING, 1978 01:17:02,560 --> 01:17:04,080 ESPECIALLY IN THE RARE DISEASE 1979 01:17:04,080 --> 01:17:04,280 SPACE. 1980 01:17:04,280 --> 01:17:06,600 YOU'RE NOT GOING TO GET TWO, 1981 01:17:06,600 --> 01:17:09,000 THREE TRIES AT DOING THIS. 1982 01:17:09,000 --> 01:17:11,080 AND SO IT'S IMPORTANT TO HAVE 1983 01:17:11,080 --> 01:17:13,280 YOUR VOICE HEARD SO THAT THE 1984 01:17:13,280 --> 01:17:15,440 DEVELOPERS ARE AWARE OF WHAT IT 1985 01:17:15,440 --> 01:17:18,320 IS THAT PATIENT GROUPS VIEW TO 1986 01:17:18,320 --> 01:17:19,600 BE IMPORTANT AND DESIGN THEIR 1987 01:17:19,600 --> 01:17:21,360 CLINICAL TRIALS WITH THAT IN 1988 01:17:21,360 --> 01:17:23,000 MIND. 1989 01:17:23,000 --> 01:17:23,240 THANKS. 1990 01:17:23,240 --> 01:17:26,000 >> THANK YOU SO MUCH. 1991 01:17:26,000 --> 01:17:28,880 I'D LIKE TO CONCLUDE THAT 1992 01:17:28,880 --> 01:17:32,800 QUESTION BY SAYING WHAT I OFTEN 1993 01:17:32,800 --> 01:17:34,680 SAY TO OTHER ADVOCATES. 1994 01:17:34,680 --> 01:17:38,280 WE'VE HEARD ABOUT WHAT WE CAN DO 1995 01:17:38,280 --> 01:17:40,040 NOW TO PREPARE OUR COMMUNITIES 1996 01:17:40,040 --> 01:17:44,000 FOR GENE THERAPY AND GENE 1997 01:17:44,000 --> 01:17:44,480 EDITING. 1998 01:17:44,480 --> 01:17:47,160 YOU'VE HEARD ABOUT CONTACT 1999 01:17:47,160 --> 01:17:49,480 REGISTRIES, NATURAL HISTORY 2000 01:17:49,480 --> 01:17:52,520 STUDIES, CLINICAL OUTCOME 2001 01:17:52,520 --> 01:17:52,760 MEASURES. 2002 01:17:52,760 --> 01:17:55,080 MY FINAL ENCOURAGEMENT IS NEVER 2003 01:17:55,080 --> 01:17:56,040 GET INTIMIDATED. 2004 01:17:56,040 --> 01:17:57,240 GET STARTED. 2005 01:17:57,240 --> 01:17:59,600 YOU KNOW, WE ALL STARTED ON THE 2006 01:17:59,600 --> 01:18:02,160 KITCHEN TABLE WITH ALL THESE 2007 01:18:02,160 --> 01:18:02,400 THINGS. 2008 01:18:02,400 --> 01:18:04,240 NOW WE'VE GOT WORLD CLASS 2009 01:18:04,240 --> 01:18:05,560 NATURAL HISTORY STUDIES THAT ARE 2010 01:18:05,560 --> 01:18:06,680 MAKING A HUGE DIFFERENCE WITH 2011 01:18:06,680 --> 01:18:12,040 OUR FRIENDS AT THE FDA. 2012 01:18:12,040 --> 01:18:14,200 WE'VE GOT CONTACT REGISTRIES 2013 01:18:14,200 --> 01:18:15,600 THAT ARE ENROLLING CLINICAL 2014 01:18:15,600 --> 01:18:17,920 TRIALS IN A MATTER OF HOURS 2015 01:18:17,920 --> 01:18:22,560 RATHER THAN WEEKS, MONTHS, 2016 01:18:22,560 --> 01:18:22,880 YEARS. 2017 01:18:22,880 --> 01:18:24,600 IF YOU NEED THEM DON'T BE 2018 01:18:24,600 --> 01:18:26,600 INTIMIDATED BY THE FINAL PRODUCT 2019 01:18:26,600 --> 01:18:28,760 THAT'S TAKEN US 25 YEARS TO 2020 01:18:28,760 --> 01:18:29,000 DEVELOP. 2021 01:18:29,000 --> 01:18:29,760 GET STARTED. 2022 01:18:29,760 --> 01:18:35,800 EVEN IF IT'S AT THE KITCHEN 2023 01:18:35,800 --> 01:18:36,000 TABLE. 2024 01:18:36,000 --> 01:18:37,440 >> RON, CAN I SAY ONE THING 2025 01:18:37,440 --> 01:18:38,440 ABOUT THAT? 2026 01:18:38,440 --> 01:18:41,040 WE HAVE NEW GROUPS COMING IN, 2027 01:18:41,040 --> 01:18:42,680 THEY WANT TO NOW WHAT CAN WE DO 2028 01:18:42,680 --> 01:18:46,080 AND HOW DO WE DO THIS, WE SAY 2029 01:18:46,080 --> 01:18:50,560 ASK RON BARTEK AND SEE WHAT HE 2030 01:18:50,560 --> 01:18:51,200 DID. 2031 01:18:51,200 --> 01:18:52,640 BECAUSE FARA KNOWS HOW TO DO IT, 2032 01:18:52,640 --> 01:18:54,920 LEARN FROM THEM. 2033 01:18:54,920 --> 01:18:58,080 >> YOU WERE DETERMINED TO MAKE 2034 01:18:58,080 --> 01:18:59,280 ME CRY, WEREN'T YOU, PJ? 2035 01:18:59,280 --> 01:19:03,160 >> NO, NO, IT'S TRUE. 2036 01:19:03,160 --> 01:19:03,360 RIGHT? 2037 01:19:03,360 --> 01:19:04,160 >> THANK YOU. 2038 01:19:04,160 --> 01:19:07,320 DIANA, DO WE HAVE ANY QUESTIONS 2039 01:19:07,320 --> 01:19:07,680 ONLINE? 2040 01:19:07,680 --> 01:19:08,800 NOT YET, OKAY. 2041 01:19:08,800 --> 01:19:10,880 PLEASE. 2042 01:19:10,880 --> 01:19:12,480 TERRY, GO. 2043 01:19:12,480 --> 01:19:13,600 YOU GOT THE MIC. 2044 01:19:13,600 --> 01:19:15,040 >> THESE ARE NOT QUESTIONS. 2045 01:19:15,040 --> 01:19:17,080 ONE MIGHT BE A QUESTION. 2046 01:19:17,080 --> 01:19:18,960 I'M GOING TO CALL A FEW THINGS 2047 01:19:18,960 --> 01:19:19,200 OUT. 2048 01:19:19,200 --> 01:19:21,040 I WANT TO ADDRESS THAT THE GENE 2049 01:19:21,040 --> 01:19:27,960 THERAPIES THAT HAVE BEEN MADE 2050 01:19:27,960 --> 01:19:29,720 TODAY, ADC DEFICIENCY, SMA, 2051 01:19:29,720 --> 01:19:31,000 CHERRY PICKED DISEASES, DECADES 2052 01:19:31,000 --> 01:19:34,320 IN THE MAKING, SHOWING AMAZING 2053 01:19:34,320 --> 01:19:34,560 PROGRESS. 2054 01:19:34,560 --> 01:19:36,400 GENE THERAPIES LIKE FOR MY SON 2055 01:19:36,400 --> 01:19:38,360 THAT WE'RE MAKING NOW, THESE ARE 2056 01:19:38,360 --> 01:19:39,640 VERY COMPLEX DISEASES. 2057 01:19:39,640 --> 01:19:41,280 IF WE'RE LUCKY WE'LL SLOW DOWN 2058 01:19:41,280 --> 01:19:42,160 THE DISEASE. 2059 01:19:42,160 --> 01:19:44,120 WE'RE NOT GOING TO SEE THESE 2060 01:19:44,120 --> 01:19:45,880 KIDS WALKING AND TALKING WHEN 2061 01:19:45,880 --> 01:19:47,760 THE GENERATION LIKE DR. BROOKS 2062 01:19:47,760 --> 01:19:48,640 SAID IS ALREADY DONE. 2063 01:19:48,640 --> 01:19:53,880 WE HAVE TO CATCH THESE KIDS AT 3 2064 01:19:53,880 --> 01:19:56,160 MONTHS, 6 MONTHS, BEFORE 2065 01:19:56,160 --> 01:19:57,080 MICROCEPHALY KICKS IN. 2066 01:19:57,080 --> 01:19:58,720 HOW DO WE DO THAT? 2067 01:19:58,720 --> 01:20:01,200 IF A DRUG IS IN THE CLINICAL 2068 01:20:01,200 --> 01:20:02,840 TRIAL, MAYBE THAT'S WHEN THE 2069 01:20:02,840 --> 01:20:06,080 HEEL PRICK IS THE NEWBORN 2070 01:20:06,080 --> 01:20:07,560 SCREENING PROGRAM IS KICKED OFF, 2071 01:20:07,560 --> 01:20:10,200 NOT AFTER THE DRUG IS APPROVED. 2072 01:20:10,200 --> 01:20:13,000 WE HAVE TO FIGURE OUT HOW TO GET 2073 01:20:13,000 --> 01:20:15,040 INSURANCE COMPANIES TO PAY FOR 2074 01:20:15,040 --> 01:20:16,440 ASOs AND GENE THERAPIES, THAT 2075 01:20:16,440 --> 01:20:18,000 KIND OF STUFF, WHILE THEY ARE IN 2076 01:20:18,000 --> 01:20:19,640 THE CLINIC, NOT AFTER THEY ARE 2077 01:20:19,640 --> 01:20:19,920 APPROVED. 2078 01:20:19,920 --> 01:20:21,720 I THINK THAT'S WHERE WE'RE GOING 2079 01:20:21,720 --> 01:20:24,240 TO MAKE A BIGGER IMPACT. 2080 01:20:24,240 --> 01:20:26,640 INSTEAD OF DOSING 10, 20, 100 2081 01:20:26,640 --> 01:20:27,200 PATIENTS, WE COULD DOSE 2082 01:20:27,200 --> 01:20:29,600 THOUSANDS AND SEE IF IT REALLY 2083 01:20:29,600 --> 01:20:32,120 WORKS AND MOVE THINGS FORWARD. 2084 01:20:32,120 --> 01:20:34,880 I KNOW THAT WE'RE SEEING AMAZING 2085 01:20:34,880 --> 01:20:36,520 THINGS HAPPENING BUT I THINK IF 2086 01:20:36,520 --> 01:20:38,160 YOU WENT TO A PARENT AND SAID I 2087 01:20:38,160 --> 01:20:39,360 CAN GIVE YOU FIVE YEARS OF 2088 01:20:39,360 --> 01:20:40,920 BETTER LIFE FOR YOUR CHILD, 2089 01:20:40,920 --> 01:20:43,400 THAT'S A WIN. 2090 01:20:43,400 --> 01:20:44,720 THAT'S A HUGE WIN. 2091 01:20:44,720 --> 01:20:48,360 OR MY CHILD IS ABLE TO CRAWL FOR 2092 01:20:48,360 --> 01:20:49,120 FIVE MORE YEARS. 2093 01:20:49,120 --> 01:20:51,760 OR ABLE TO GO TO BED WITHOUT 2094 01:20:51,760 --> 01:20:52,440 PAIN. 2095 01:20:52,440 --> 01:20:53,400 THESE ARE BIG WINS. 2096 01:20:53,400 --> 01:20:55,160 WHEN WE LOOK AT THEM, WE THINK 2097 01:20:55,160 --> 01:20:56,160 THEY ARE SMALL. 2098 01:20:56,160 --> 01:21:01,160 BUT FOR FOR A PARENT IT'S HUGE. 2099 01:21:01,160 --> 01:21:03,280 THESE ARE THE THINGS WE HAVE TO 2100 01:21:03,280 --> 01:21:05,000 THINK OF, MAYBE SMALL WINS TO 2101 01:21:05,000 --> 01:21:06,160 PEOPLE OUTSIDE THE COMMUNITY BUT 2102 01:21:06,160 --> 01:21:11,360 HUGE WINS FOR ALL OF US. 2103 01:21:11,360 --> 01:21:12,640 >> ABSOLUTELY. 2104 01:21:12,640 --> 01:21:13,320 NEXT QUESTION? 2105 01:21:13,320 --> 01:21:15,760 COULD EACH PERSON WITH A 2106 01:21:15,760 --> 01:21:19,880 QUESTION IDENTIFY THEMSELVES 2107 01:21:19,880 --> 01:21:20,080 FIRST. 2108 01:21:20,080 --> 01:21:24,400 >> HI. 2109 01:21:24,400 --> 01:21:28,640 CAITLIN ESPOSITO, WITH BOBBY 2110 01:21:28,640 --> 01:21:30,680 JONES. 2111 01:21:30,680 --> 01:21:33,440 AS FAR AS MANUFACTURING AND 2112 01:21:33,440 --> 01:21:35,840 SCALING, IS THERE ANY FUNDING 2113 01:21:35,840 --> 01:21:36,480 OPPORTUNITIES AVAILABLE RIGHT 2114 01:21:36,480 --> 01:21:40,760 NOW OR WHO IS FUNDING THOSE 2115 01:21:40,760 --> 01:21:41,320 INNOVATIONS? 2116 01:21:41,320 --> 01:21:43,480 AND IF SO, WHO IS, MORE 2117 01:21:43,480 --> 01:21:44,480 IMPORTANTLY, MONITORING THAT, 2118 01:21:44,480 --> 01:21:48,520 AND IS THAT A POTENTIAL ROLE FOR 2119 01:21:48,520 --> 01:21:52,120 PATIENT ADVOCACY ORGANIZATIONS, 2120 01:21:52,120 --> 01:21:53,440 MAYBE SOMEHOW INVOLVED IN 2121 01:21:53,440 --> 01:21:54,880 MONITORING OF THAT INNOVATION 2122 01:21:54,880 --> 01:21:58,160 AND SCALE AND MANUFACTURING 2123 01:21:58,160 --> 01:22:01,880 PURPOSES? 2124 01:22:01,880 --> 01:22:05,520 >> MAYBE GOPA AND P.J. WOULD 2125 01:22:05,520 --> 01:22:07,120 LIKE TO WEIGH IN. 2126 01:22:07,120 --> 01:22:09,920 >> YEAH, I'LL GO. 2127 01:22:09,920 --> 01:22:12,280 WE ACTUALLY DO FUND -- WE ARE 2128 01:22:12,280 --> 01:22:17,760 FUNDING ONE PROJECT TO THE SMALL 2129 01:22:17,760 --> 01:22:18,960 BUSINESS PROGRAM THAT AIMS TO 2130 01:22:18,960 --> 01:22:20,720 FIND A MORE EFFICIENT WAY TO 2131 01:22:20,720 --> 01:22:23,360 GROW THE CELLS TO MAKE AAV GENE 2132 01:22:23,360 --> 01:22:24,000 THERAPY. 2133 01:22:24,000 --> 01:22:27,320 SO WE ARE ABLE TO DO THAT. 2134 01:22:27,320 --> 01:22:30,280 I THINK THAT -- BUT GOPA ALSO -- 2135 01:22:30,280 --> 01:22:31,360 THEY FUND WORK ALONG THOSE LINES 2136 01:22:31,360 --> 01:22:34,440 AT FDA TOO. 2137 01:22:34,440 --> 01:22:36,800 >> YES, SO THANKS FOR THE 2138 01:22:36,800 --> 01:22:37,360 QUESTION. 2139 01:22:37,360 --> 01:22:37,960 YES, THAT'S CRITICALLY 2140 01:22:37,960 --> 01:22:38,240 IMPORTANT. 2141 01:22:38,240 --> 01:22:41,360 ONE OF THE ISSUES WE HAVE RIGHT 2142 01:22:41,360 --> 01:22:44,320 NOW IS THAT MANUFACTURING IS A 2143 01:22:44,320 --> 01:22:44,920 ROAD BLOCK. 2144 01:22:44,920 --> 01:22:47,440 THERE JUST ISN'T THE 2145 01:22:47,440 --> 01:22:50,160 MANUFACTURING CAPACITY TO MAKE 2146 01:22:50,160 --> 01:22:51,160 AAV GENE THERAPIES, FOR EXAMPLE, 2147 01:22:51,160 --> 01:22:53,040 IN LARGE SCALES FOR LARGE 2148 01:22:53,040 --> 01:22:53,360 POPULATIONS. 2149 01:22:53,360 --> 01:22:54,560 IT'S A PROBLEM AT THE OTHER END 2150 01:22:54,560 --> 01:22:58,200 ALSO IN TERMS OF COST. 2151 01:22:58,200 --> 01:23:00,480 SO THE TWO APPROACHES, AND BGTC 2152 01:23:00,480 --> 01:23:03,320 IS FUNDING THIS ALSO AS PART OF 2153 01:23:03,320 --> 01:23:06,040 THEIR RESEARCH COMPONENT, WHICH 2154 01:23:06,040 --> 01:23:08,680 IS TO SEE MORE EFFICIENT MEANS 2155 01:23:08,680 --> 01:23:10,000 OF MANUFACTURING AAV GENE 2156 01:23:10,000 --> 01:23:12,080 THERAPIES AS WELL AS ENHANCING 2157 01:23:12,080 --> 01:23:12,720 GENE EXPRESSION. 2158 01:23:12,720 --> 01:23:15,680 WE AT FDA ALSO HAVE PUT OUT 2159 01:23:15,680 --> 01:23:16,080 GRANTS. 2160 01:23:16,080 --> 01:23:18,680 THE LAST ONE WE PUT OUT FUNDED 2161 01:23:18,680 --> 01:23:21,920 MAYBE FIVE OR SIX DIFFERENT 2162 01:23:21,920 --> 01:23:24,800 RESEARCH PROJECTS SPECIFICALLY 2163 01:23:24,800 --> 01:23:27,400 FOCUSED ON ENHANCING AAV GENE 2164 01:23:27,400 --> 01:23:29,280 THERAPIES FOR VERY SMALL 2165 01:23:29,280 --> 01:23:32,000 POPULATIONS, LOOKING AT 2166 01:23:32,000 --> 01:23:32,680 OPPORTUNITIES TO INCREASE 2167 01:23:32,680 --> 01:23:33,800 EFFICIENCY OF THE MANUFACTURING. 2168 01:23:33,800 --> 01:23:35,080 YOU RAISE THE POINT I JUST 2169 01:23:35,080 --> 01:23:36,360 WANTED TO TAKE THE OPPORTUNITY 2170 01:23:36,360 --> 01:23:38,600 TO TOUCH ON WHEN YOU THINK ABOUT 2171 01:23:38,600 --> 01:23:39,480 PRODUCT DEVELOPMENT WHICH IS 2172 01:23:39,480 --> 01:23:42,760 THERE'S A LOT OF FOCUS ON 2173 01:23:42,760 --> 01:23:43,680 CLINICAL DEVELOPMENT. 2174 01:23:43,680 --> 01:23:45,920 THERE SHOULD BE. 2175 01:23:45,920 --> 01:23:47,480 BUT USUALLY WHAT ENDS UP BEING A 2176 01:23:47,480 --> 01:23:50,960 PROBLEM AT THE END OF THE DAY IS 2177 01:23:50,960 --> 01:23:53,280 MANUFACTURING, IN TERMS OF 2178 01:23:53,280 --> 01:23:54,120 PRODUCT APPROVAL. 2179 01:23:54,120 --> 01:23:55,520 SO, AGAIN, THAT'S SOMETHING ONE 2180 01:23:55,520 --> 01:23:58,320 NEEDS TO BE FORWARD THINKING ON. 2181 01:23:58,320 --> 01:23:59,720 IF YOU'RE DOING SMALL SCALE 2182 01:23:59,720 --> 01:24:01,440 MANUFACTURING FOR EARLY PHASE 2183 01:24:01,440 --> 01:24:02,560 CLINICAL TRIALS, HOW ARE YOU 2184 01:24:02,560 --> 01:24:06,480 GOING TO SCALE UP AND DO THE 2185 01:24:06,480 --> 01:24:07,680 COMPARABILITY, IF YOUR PATIENT 2186 01:24:07,680 --> 01:24:10,080 POPULATION IS ONLY TEN PEOPLE 2187 01:24:10,080 --> 01:24:11,720 MAYBE THERE'S SOME MEANS TO DO A 2188 01:24:11,720 --> 01:24:13,720 BATCH SUCH THAT YOU DO THE 2189 01:24:13,720 --> 01:24:15,840 PRE-CLINICAL TESTING AS WELL AS 2190 01:24:15,840 --> 01:24:16,800 THE CLINICAL TESTING USING THE 2191 01:24:16,800 --> 01:24:17,680 SAME BATCH. 2192 01:24:17,680 --> 01:24:21,720 YOU HAVE TO THINK ABOUT -- COST 2193 01:24:21,720 --> 01:24:24,240 FACTOR COMMENTS IN BUT I THINK 2194 01:24:24,240 --> 01:24:25,920 IN THE RARE DISEASE SPACE 2195 01:24:25,920 --> 01:24:27,320 THINKING AHEAD WHERE DO YOU WANT 2196 01:24:27,320 --> 01:24:28,880 TO END UP, AND THEN WORK 2197 01:24:28,880 --> 01:24:30,000 BACKWARDS TO SEE WHAT YOU NEED 2198 01:24:30,000 --> 01:24:33,920 TO DO TO GET THERE MOST 2199 01:24:33,920 --> 01:24:34,440 EFFICIENTLY IS IMPORTANT. 2200 01:24:34,440 --> 01:24:38,200 >> I WANT TO ADD ONE OTHER 2201 01:24:38,200 --> 01:24:38,400 THING. 2202 01:24:38,400 --> 01:24:39,640 GOPA TOUCHED ON THE RESEARCH THE 2203 01:24:39,640 --> 01:24:41,800 BGTC IS FUNDING WHICH WE HOPE 2204 01:24:41,800 --> 01:24:43,160 WILL EVENTUALLY LEAD TO 2205 01:24:43,160 --> 01:24:44,440 IMPROVEMENTS IN THE 2206 01:24:44,440 --> 01:24:45,440 MANUFACTURING PROCESS. 2207 01:24:45,440 --> 01:24:48,200 ONE OTHER SPACE TO WATCH, THERE 2208 01:24:48,200 --> 01:24:50,800 IS A MANUFACTURING USA 2209 01:24:50,800 --> 01:24:54,320 INSTITUTE, NATIONAL INSTITUTE 2210 01:24:54,320 --> 01:24:56,400 FOR INNOVATION MANUFACTURING 2211 01:24:56,400 --> 01:24:57,800 BIOPHARMACEUTICALS, NIMBLE WITH 2212 01:24:57,800 --> 01:24:59,040 TWO Is. 2213 01:24:59,040 --> 01:25:01,680 THEY ARE FUNDED BY THE NATIONAL 2214 01:25:01,680 --> 01:25:03,400 INSTITUTE OF STANDARDS AND 2215 01:25:03,400 --> 01:25:04,160 TECHNOLOGY WHICH WHERE I WAS 2216 01:25:04,160 --> 01:25:06,120 BEFORE I CAME TO THE FOUNDATION, 2217 01:25:06,120 --> 01:25:09,080 A FULL CIRCLE CONNECTION THERE. 2218 01:25:09,080 --> 01:25:10,960 AS PART OF THE CONTRIBUTION TO 2219 01:25:10,960 --> 01:25:15,560 BGTC, THEY ARE DOING A VERY 2220 01:25:15,560 --> 01:25:17,280 LARGE AMOUNT OF IN-KIND 2221 01:25:17,280 --> 01:25:19,040 RESEARCH, $6 MILLION OF EFFORT 2222 01:25:19,040 --> 01:25:20,600 TOWARDS AN OPEN SOURCE 2223 01:25:20,600 --> 01:25:25,720 MANUFACTURING PLATFORM FOR GENE 2224 01:25:25,720 --> 01:25:26,480 THERAPIES. 2225 01:25:26,480 --> 01:25:28,040 THAT'S SOMETHING PROCEEDING 2226 01:25:28,040 --> 01:25:29,720 ALONGSIDE THE BGTC, BASED ON THE 2227 01:25:29,720 --> 01:25:30,720 TIMING OF THE WORK, WASN'T 2228 01:25:30,720 --> 01:25:32,800 SOMETHING WE COULD INCORPORATE 2229 01:25:32,800 --> 01:25:33,920 INTO OUR MANUFACTURING 2230 01:25:33,920 --> 01:25:36,320 PRE-CLINICAL TESTING BUT COULD 2231 01:25:36,320 --> 01:25:38,960 POTENTIALLY FORM THE BASIS OF A 2232 01:25:38,960 --> 01:25:40,480 FUTURE ITERATION OF A 2233 01:25:40,480 --> 01:25:42,040 CONSORTIUM, ALL THE AMP PROGRAMS 2234 01:25:42,040 --> 01:25:45,960 THAT STARTED IN 2014 HAVE SINCE 2235 01:25:45,960 --> 01:25:48,600 CONTINUED ON OR EXPANDED INTO 2236 01:25:48,600 --> 01:25:50,000 LARGER PROGRAMS OR BROADER 2237 01:25:50,000 --> 01:25:50,240 PROGRAMS. 2238 01:25:50,240 --> 01:25:53,720 I THINK THERE'S GOING TO BE A 2239 01:25:53,720 --> 01:25:55,240 LOT OF EXPECTATION THE BGTC WILL 2240 01:25:55,240 --> 01:25:56,800 DO SO ALSO. 2241 01:25:56,800 --> 01:25:58,880 SO OVER THE NEXT TWO TO THREE 2242 01:25:58,880 --> 01:26:05,520 YEARS NIMBLE WILL BE PUTTING OUT 2243 01:26:05,520 --> 01:26:06,320 PROTOCOLS, VARIOUS RESEARCH 2244 01:26:06,320 --> 01:26:07,520 RESOURCES, REFERENCE MATERIALS 2245 01:26:07,520 --> 01:26:08,920 AND STANDARDS THAT WILL ALSO 2246 01:26:08,920 --> 01:26:13,120 REALLY HELP MOVE TOWARDS THAT 2247 01:26:13,120 --> 01:26:13,960 PLATFORM AND MANUFACTURING 2248 01:26:13,960 --> 01:26:14,200 PROCESS. 2249 01:26:14,200 --> 01:26:16,560 >> RON, I CAN FOLLOW UP WITH ONE 2250 01:26:16,560 --> 01:26:19,120 MORE POINT, THAT THE QUESTION 2251 01:26:19,120 --> 01:26:21,200 ABOUT MANUFACTURING FOR GENE 2252 01:26:21,200 --> 01:26:22,960 THERAPIES CAN BE QUITE DIFFERENT 2253 01:26:22,960 --> 01:26:25,360 THAN GENE EDITING. 2254 01:26:25,360 --> 01:26:28,640 FOR GENE THERAPY, USING AAV, YOU 2255 01:26:28,640 --> 01:26:30,720 NEED TO KEEP -- TO GET AAV IN 2256 01:26:30,720 --> 01:26:33,680 THE CELL TO KEEP MAKING THE 2257 01:26:33,680 --> 01:26:35,000 THERAPY TO PROTEIN. 2258 01:26:35,000 --> 01:26:36,400 FOR GENE EDITING IT'S ACTUALLY 2259 01:26:36,400 --> 01:26:37,320 QUITE THE OPPOSITE. 2260 01:26:37,320 --> 01:26:41,000 YOU NEED TO GET A GENOME EDITOR 2261 01:26:41,000 --> 01:26:44,520 TO CORRECT THE MUTATION BUT ONCE 2262 01:26:44,520 --> 01:26:45,600 THAT'S DONE YOU WOULD RATHER 2263 01:26:45,600 --> 01:26:47,040 HAVE IT GO AWAY. 2264 01:26:47,040 --> 01:26:48,680 THAT'S ONE OF THE THINGS WE'RE 2265 01:26:48,680 --> 01:26:51,120 EXCITED ABOUT IN THE GENOME 2266 01:26:51,120 --> 01:26:52,560 EDITING PROGRAM IS TO SEE IF WE 2267 01:26:52,560 --> 01:26:57,800 CAN ADAPT THE SAME TECHNOLOGY 2268 01:26:57,800 --> 01:27:00,160 USED IN THE COVID VACCINES AND 2269 01:27:00,160 --> 01:27:01,920 ADAPT THAT FOR THE DELIVERY OF 2270 01:27:01,920 --> 01:27:05,440 GENOME EDITORS. 2271 01:27:05,440 --> 01:27:09,400 SO WE'LL CALL IT A SIMPLE 2272 01:27:09,400 --> 01:27:11,040 NANOPARTICLE, NOT SIMPLE TO 2273 01:27:11,040 --> 01:27:12,880 CROSS THE BLOOD-BRAIN BARRIER, 2274 01:27:12,880 --> 01:27:15,520 BUT THAT'S THE IDEA, A QUITE 2275 01:27:15,520 --> 01:27:16,720 DIFFERENT CHALLENGING FOR GENOME 2276 01:27:16,720 --> 01:27:19,040 EDITING AND GENE THERAPY. 2277 01:27:19,040 --> 01:27:20,560 SPEAKING FROM NCATS WE'RE TRYING 2278 01:27:20,560 --> 01:27:23,480 TO PLAY BOTH SIDES OF THE STREET 2279 01:27:23,480 --> 01:27:24,040 HERE. 2280 01:27:24,040 --> 01:27:25,880 BECAUSE I THINK WE WANT AS MANY 2281 01:27:25,880 --> 01:27:28,240 SHOTS ON GOAL AS POSSIBLE. 2282 01:27:28,240 --> 01:27:33,800 >> I WOULD LIKE TO ADD TO THIS 2283 01:27:33,800 --> 01:27:35,240 DISCUSSION OF MANUFACTURING, 2284 01:27:35,240 --> 01:27:38,320 GOPA, YOU MENTIONED EARLIER DR. 2285 01:27:38,320 --> 01:27:40,040 PETER MARX, ONE OF THE TRUE 2286 01:27:40,040 --> 01:27:46,720 LEADERS IN THIS FIELD, TRYING O 2287 01:27:46,720 --> 01:27:49,480 INCREASE EFFICIENCY AND REDUCE 2288 01:27:49,480 --> 01:27:50,560 COST OF MANUFACTURING, THE 2289 01:27:50,560 --> 01:27:51,880 LONGEST POLE IN THE TENT. 2290 01:27:51,880 --> 01:27:55,080 IT MAKES GENE THERAPIES SO 2291 01:27:55,080 --> 01:27:57,920 OUTRAGEOUSLY -- I SHOULDN'T SAY 2292 01:27:57,920 --> 01:28:01,280 OUTRAGEOUSLY EXPENSIVE BUT IT'S 2293 01:28:01,280 --> 01:28:06,240 A KEY PLAYER. 2294 01:28:06,240 --> 01:28:07,560 COURTNEY MENTIONED NIIMBLE. 2295 01:28:07,560 --> 01:28:11,040 TWO YEARS AGO THEY HELD THEIR 2296 01:28:11,040 --> 01:28:12,280 ANNUAL SUMMIT, PETER MARKS AND 2297 01:28:12,280 --> 01:28:16,080 P.J. AND I SPOKE ABOUT THE 2298 01:28:16,080 --> 01:28:21,680 IMPORTANCE OF INCREASING 2299 01:28:21,680 --> 01:28:22,480 EFFICIENCY, REDUCING COST, 2300 01:28:22,480 --> 01:28:23,320 MANUFACTURING, TWO YEARS AGO, 2301 01:28:23,320 --> 01:28:28,560 AND IN THIS ROOM WE HAD AN NCATS 2302 01:28:28,560 --> 01:28:34,400 WORKSHOP ON MANUFACTURING WHICH 2303 01:28:34,400 --> 01:28:35,800 THE NCATS DIRECTOR AND PETER 2304 01:28:35,800 --> 01:28:41,600 MARKS CHAIRED THE DISCUSSION, WE 2305 01:28:41,600 --> 01:28:43,600 HAD INDUSTRY PARTNERS, ACADEMIC 2306 01:28:43,600 --> 01:28:44,080 INVESTIGATORS, ADVOCATES 2307 01:28:44,080 --> 01:28:45,480 DISCUSSING THIS ISSUE. 2308 01:28:45,480 --> 01:28:46,640 WE'VE COME A LONG WAY. 2309 01:28:46,640 --> 01:28:49,520 WE'VE GOT A LONG WAY TO GO. 2310 01:28:49,520 --> 01:28:57,960 I THINK DIANA HAS A QUESTION. 2311 01:28:57,960 --> 01:28:58,360 >> THANKS, RON. 2312 01:28:58,360 --> 01:29:03,080 I HAVE A COMMENT FROM HEIDI 2313 01:29:03,080 --> 01:29:05,720 WALLIS, BUT IT MIGHT PROMPT 2314 01:29:05,720 --> 01:29:07,800 DISCUSSION OF ADVOCACY IN THIS 2315 01:29:07,800 --> 01:29:08,000 AREA. 2316 01:29:08,000 --> 01:29:10,080 IN ADDITION TO ASKING INSURANCE 2317 01:29:10,080 --> 01:29:13,720 TO COVER EXPENSIVE DRUGS, WE 2318 01:29:13,720 --> 01:29:17,240 NEED TO CONQUER GET NEWBORN 2319 01:29:17,240 --> 01:29:18,680 SCREENING PAID FOR. 2320 01:29:18,680 --> 01:29:21,160 SO MANY STATES ARE UNDERFUNDED 2321 01:29:21,160 --> 01:29:22,440 IN THIS CRITICAL PROGRAM. 2322 01:29:22,440 --> 01:29:23,720 >> WHO WOULD LIKE TO TAKE 2323 01:29:23,720 --> 01:29:30,560 NEWBORN SCREENING ON? 2324 01:29:30,560 --> 01:29:33,120 >> I MEAN, I THINK THERE'S 2325 01:29:33,120 --> 01:29:34,520 CERTAINLY MANY, MANY CHALLENGES 2326 01:29:34,520 --> 01:29:34,880 THERE. 2327 01:29:34,880 --> 01:29:39,200 I DO THINK THAT IT RELATES TO -- 2328 01:29:39,200 --> 01:29:40,320 I SAY NEWBORN SCREENING, LET ME 2329 01:29:40,320 --> 01:29:41,840 BACK UP AND SAY THIS IS MY 2330 01:29:41,840 --> 01:29:43,240 PERSONAL OPINION HERE. 2331 01:29:43,240 --> 01:29:46,360 I DO THINK THAT THE NEWBORN 2332 01:29:46,360 --> 01:29:47,840 SCREENING FIELD NEEDS TO TAKE 2333 01:29:47,840 --> 01:29:51,680 INTO ACCOUNT THE FACT THAT WE'RE 2334 01:29:51,680 --> 01:29:54,080 STARTING TO DEVELOP GENE TARGET 2335 01:29:54,080 --> 01:29:55,520 THERAPIES AS THERAPEUTIC 2336 01:29:55,520 --> 01:29:57,600 PLATFORMS, WHAT AS CURRENTLY 2337 01:29:57,600 --> 01:29:59,120 NEWBORN SCREENING IS ONE DISEASE 2338 01:29:59,120 --> 01:30:00,400 AT A TIME, ANOTHER EXAMPLE OF 2339 01:30:00,400 --> 01:30:03,160 THE SQUARE PEG IN THE ROUND HOLE 2340 01:30:03,160 --> 01:30:03,520 SITUATION. 2341 01:30:03,520 --> 01:30:06,560 A COUPLE YEARS AGO WE HAD A 2342 01:30:06,560 --> 01:30:11,840 SERIES OF MEETINGS SPONSORED BY 2343 01:30:11,840 --> 01:30:14,440 NCATS TO TRY TO DEAL WITH THAT 2344 01:30:14,440 --> 01:30:15,360 LACK OF CONVERGENCE. 2345 01:30:15,360 --> 01:30:17,520 AND I THINK THAT REMAINS A 2346 01:30:17,520 --> 01:30:17,760 PROBLEM. 2347 01:30:17,760 --> 01:30:21,240 I'M GOING TO PUT INTO THE CHAT 2348 01:30:21,240 --> 01:30:22,640 SOME INFORMATION ABOUT THOSE 2349 01:30:22,640 --> 01:30:24,160 SERIES OF MEETINGS. 2350 01:30:24,160 --> 01:30:29,040 I WOULD AGREE, I THINK THERE'S A 2351 01:30:29,040 --> 01:30:30,600 MAJOR NEED FOR REEVALUATION OF 2352 01:30:30,600 --> 01:30:31,760 THE WAY WE DO NEWBORN SCREENING 2353 01:30:31,760 --> 01:30:35,520 IN LIGHT OF THE WAY WE'RE 2354 01:30:35,520 --> 01:30:36,480 DEVELOPING GENETIC THERAPIES 2355 01:30:36,480 --> 01:30:41,720 NOW, NOT JUST GENE THERAPY AND 2356 01:30:41,720 --> 01:30:42,960 GENE EDITING BUT 2357 01:30:42,960 --> 01:30:45,120 OLIGONUCLEOTIDES, THE SPEAK YOU 2358 01:30:45,120 --> 01:30:47,400 CAN DEVELOP A THERAPEUTIC FOR A 2359 01:30:47,400 --> 01:30:50,240 DISEASE NOW IS VASTLY DIFFERENT 2360 01:30:50,240 --> 01:30:54,080 THAN WHEN THE NEWBORN SCREENING 2361 01:30:54,080 --> 01:30:55,880 LEGISLATION WAS WRITTEN I THINK 2362 01:30:55,880 --> 01:30:57,080 IN THE 1980s AND THERE HAS TO 2363 01:30:57,080 --> 01:30:59,280 BE A CONSIDERATION OF THAT. 2364 01:30:59,280 --> 01:31:02,120 >> I'LL PILE ONTO THAT, P.J., 2365 01:31:02,120 --> 01:31:06,160 AND SAY FOR MANY OF US IN THE 2366 01:31:06,160 --> 01:31:09,000 ADVOCACY COMMUNITY, WE SEE THE 2367 01:31:09,000 --> 01:31:10,680 CRYING NEED FOR EARLIER 2368 01:31:10,680 --> 01:31:11,280 IDENTIFICATION. 2369 01:31:11,280 --> 01:31:13,640 TERRY MENTIONED IT IN HIS 2370 01:31:13,640 --> 01:31:13,880 DISEASE. 2371 01:31:13,880 --> 01:31:16,200 EARLIER IDENTIFICATION OF OUR 2372 01:31:16,200 --> 01:31:18,120 PATIENTS BECAUSE BY THE TIME 2373 01:31:18,120 --> 01:31:19,000 THEY ARE CLINICAL MANIFEST, 2374 01:31:19,000 --> 01:31:22,400 WHICH IS THE ONLY ALTERNATIVE TO 2375 01:31:22,400 --> 01:31:23,000 IDENTIFICATION OUTSIDE NEWBORN 2376 01:31:23,000 --> 01:31:24,440 SCREENING, SO MUCH OF THE DAMAGE 2377 01:31:24,440 --> 01:31:26,760 MIGHT HAVE BEEN DONE THAT IT'S 2378 01:31:26,760 --> 01:31:29,720 THAT MUCH MORE DIFFICULT, MAYBE 2379 01:31:29,720 --> 01:31:32,360 EVEN IMPOSSIBLE TO REVERSE. 2380 01:31:32,360 --> 01:31:35,400 SO I AGREE WE NEED TO REVISIT 2381 01:31:35,400 --> 01:31:37,920 THE WHOLE CONCEPT OF NEWBORN 2382 01:31:37,920 --> 01:31:41,200 SCREENING, AND I KNOW IT'S BEING 2383 01:31:41,200 --> 01:31:42,720 DONE, BUT CURRENTLY YOU NOT ONLY 2384 01:31:42,720 --> 01:31:44,960 HAVE TO HAVE A TREATMENT THAT'S 2385 01:31:44,960 --> 01:31:46,680 FOR THAT DISEASE BUT YOU HAVE TO 2386 01:31:46,680 --> 01:31:48,440 HAVE A TREATMENT THAT'S BEEN 2387 01:31:48,440 --> 01:31:51,800 DEMONSTRATED TO BE NECESSARY TO 2388 01:31:51,800 --> 01:31:54,280 BE APPLIED TO INFANTS. 2389 01:31:54,280 --> 01:31:59,040 AND SO THAT'S A HIGH HURDLE. 2390 01:31:59,040 --> 01:32:01,440 UNTIL WE GET THERE, WE STILL 2391 01:32:01,440 --> 01:32:04,640 NEED TO IDENTIFY PATIENTS EARLY 2392 01:32:04,640 --> 01:32:06,760 ENOUGH TO MAKE A PROFOUND 2393 01:32:06,760 --> 01:32:10,440 BENEFIT TO THEM. 2394 01:32:10,440 --> 01:32:12,160 WHAT OTHER -- ANY OTHER 2395 01:32:12,160 --> 01:32:12,440 COMMENTS? 2396 01:32:12,440 --> 01:32:14,360 >> YEAH, I WANT TO ADD ONE 2397 01:32:14,360 --> 01:32:16,560 COMMENT TO THAT. 2398 01:32:16,560 --> 01:32:18,880 ASIDE FROM THE BENEFIT OF 2399 01:32:18,880 --> 01:32:20,520 SCREENING TOWARDS THERAPEUTIC 2400 01:32:20,520 --> 01:32:22,360 TREATMENT OPTIONS, I THINK THERE 2401 01:32:22,360 --> 01:32:24,440 MAYBE NEEDS A RECOGNITION OF THE 2402 01:32:24,440 --> 01:32:27,520 BENEFIT TO THE CAREGIVER AND THE 2403 01:32:27,520 --> 01:32:27,800 FAMILY. 2404 01:32:27,800 --> 01:32:30,160 I THINK STATISTICS ARE THAT IT 2405 01:32:30,160 --> 01:32:31,560 TAKES AN AVERAGE OF SEVEN YEARS 2406 01:32:31,560 --> 01:32:36,400 TO HAVE A RARE DISEASE DIAGNOSIS 2407 01:32:36,400 --> 01:32:37,080 CORRECTLY. 2408 01:32:37,080 --> 01:32:39,080 THAT PATIENTS TYPICALLY UNDERGO 2409 01:32:39,080 --> 01:32:40,160 MULTIPLE INCORRECT DIAGNOSES 2410 01:32:40,160 --> 01:32:42,440 ALONG THAT JOURNEY. 2411 01:32:42,440 --> 01:32:44,200 SO EVEN JUST KNOWING, NOT HAVING 2412 01:32:44,200 --> 01:32:47,800 TO GO THROUGH THAT PROCESS OF 2413 01:32:47,800 --> 01:32:49,560 FALSE DIAGNOSES AND TIME AND 2414 01:32:49,560 --> 01:32:51,080 UNCERTAINTY, I THINK IS A 2415 01:32:51,080 --> 01:32:52,840 BENEFIT TO THE FAMILY AND TO THE 2416 01:32:52,840 --> 01:32:56,000 PATIENT AND TO THEIR CAREGIVER. 2417 01:32:56,000 --> 01:32:59,200 ABSENT ANY OTHER TREATMENT 2418 01:32:59,200 --> 01:33:00,280 OPPORTUNITY. 2419 01:33:00,280 --> 01:33:01,800 >> YEAH, I AGREE WITH THAT 2420 01:33:01,800 --> 01:33:04,680 COMPLETELY, COURTNEY. 2421 01:33:04,680 --> 01:33:09,240 >> JUST ONE QUICK COMMENT AND 2422 01:33:09,240 --> 01:33:10,320 ADDED PERSPECTIVE. 2423 01:33:10,320 --> 01:33:13,200 FIRSTLY, IF YOU CAN PREVENT 2424 01:33:13,200 --> 01:33:17,800 SUFFERING, THEN YOU DO IT. 2425 01:33:17,800 --> 01:33:19,520 BUT FROM A PRODUCT DEVELOPMENT 2426 01:33:19,520 --> 01:33:21,400 SCIENTIFIC POINT OF VIEW, IF YOU 2427 01:33:21,400 --> 01:33:23,360 KNOW WHAT THE DISEASE 2428 01:33:23,360 --> 01:33:24,560 MANIFESTATION IS, YOU KNOW WHAT 2429 01:33:24,560 --> 01:33:26,560 HAPPENS TO PATIENTS WHO HAVE A 2430 01:33:26,560 --> 01:33:27,200 PARTICULAR DISEASE. 2431 01:33:27,200 --> 01:33:32,000 IF YOU CAN CATCH IT EARLY BEFORE 2432 01:33:32,000 --> 01:33:33,640 ANY OF THE MANIFESTATION STARTS 2433 01:33:33,640 --> 01:33:36,200 WHEN DO YOU THERAPEUTIC 2434 01:33:36,200 --> 01:33:38,120 TREATMENTS THE ENDPOINT IS 2435 01:33:38,120 --> 01:33:38,680 CLEAR. 2436 01:33:38,680 --> 01:33:39,440 YOU KNOW WITHOUT ANY 2437 01:33:39,440 --> 01:33:41,320 INTERVENTION YOU KNOW WHAT THE 2438 01:33:41,320 --> 01:33:42,280 OUTCOME WOULD BE. 2439 01:33:42,280 --> 01:33:45,120 IF YOU CAN INTERVENE BEFORE THE 2440 01:33:45,120 --> 01:33:46,160 SYMPTOMS START, THEN WHETHER OR 2441 01:33:46,160 --> 01:33:48,800 NOT THAT PRODUCT IS WORKING AS A 2442 01:33:48,800 --> 01:33:51,400 THERAPY WILL BECOME MUCH CLEARER 2443 01:33:51,400 --> 01:33:52,720 THAN IF DISEASE PROGRESSION HAS 2444 01:33:52,720 --> 01:33:54,720 ALREADY STARTED, AND YOU'RE JUST 2445 01:33:54,720 --> 01:33:56,200 LOOKING FOR INCREMENTAL BENEFIT. 2446 01:33:56,200 --> 01:33:58,400 THAT'S WHERE WE GET INTO 2447 01:33:58,400 --> 01:33:59,720 DIFFICULTY IN DETERMINING 2448 01:33:59,720 --> 01:34:02,800 WHETHER OR NOT A THERAPY IS 2449 01:34:02,800 --> 01:34:06,000 EFFECTIVE WHEN YOU'RE LOOKING 2450 01:34:06,000 --> 01:34:08,600 FOR INCREMENTAL CLINICAL BENEFIT 2451 01:34:08,600 --> 01:34:11,120 AS OPPOSED TO SOMETHING CLEAR 2452 01:34:11,120 --> 01:34:11,360 CUT. 2453 01:34:11,360 --> 01:34:13,880 YOU INTERVENE EARLY, YOU DON'T 2454 01:34:13,880 --> 01:34:15,720 END UP WITH DISEASE SYMPTOMS. 2455 01:34:15,720 --> 01:34:17,280 VERSUS DISEASE SYMPTOMS HAVE 2456 01:34:17,280 --> 01:34:18,600 STARTED, NOW YOU'RE LOOKING TO 2457 01:34:18,600 --> 01:34:20,880 SEE IF THERAPY IS SLOWING THINGS 2458 01:34:20,880 --> 01:34:22,480 DOWN OR BRINGING INCREMENTAL 2459 01:34:22,480 --> 01:34:22,720 BENEFIT. 2460 01:34:22,720 --> 01:34:25,600 SO THAT'S ANOTHER REASON TO 2461 01:34:25,600 --> 01:34:25,920 START EARLY. 2462 01:34:25,920 --> 01:34:30,280 >> TERRIFIC POINT. 2463 01:34:30,280 --> 01:34:31,360 TERRIFIC POINT. 2464 01:34:31,360 --> 01:34:32,360 >> CAN I ADD? 2465 01:34:32,360 --> 01:34:33,960 >> TRAVIS HAS ONE MORE COMMENT. 2466 01:34:33,960 --> 01:34:36,560 >> A QUICK COMMENT FROM THE 2467 01:34:36,560 --> 01:34:38,200 DEVELOPER SIDE, IT'S REALLY -- 2468 01:34:38,200 --> 01:34:39,400 OBVIOUSLY WE'RE NOT SURPRISED 2469 01:34:39,400 --> 01:34:41,680 YOU AGREE WITH THAT STATEMENT, 2470 01:34:41,680 --> 01:34:42,320 RIGHT? 2471 01:34:42,320 --> 01:34:43,120 BUT I THINK FIRST-IN-HUMAN 2472 01:34:43,120 --> 01:34:46,760 TRIALS WHERE THE RISK IS 2473 01:34:46,760 --> 01:34:48,560 COMPLETELY UNKNOWN, WHERE DO YOU 2474 01:34:48,560 --> 01:34:52,600 START AT FIRST PATIENT DOSING? 2475 01:34:52,600 --> 01:34:56,520 AND SO TREATING PATIENTS 2476 01:34:56,520 --> 01:34:57,280 PRESYMPTOMATICALLY WE'RE 2477 01:34:57,280 --> 01:34:58,200 INTERESTED IN DOING, THE BEST 2478 01:34:58,200 --> 01:35:03,960 WAY TO DO IT, BUT NEED TO BE 2479 01:35:03,960 --> 01:35:07,960 ABLE TO SHOW, FIRST AND 2480 01:35:07,960 --> 01:35:09,800 FOREMOST, THE PRIORITY, HAVING 2481 01:35:09,800 --> 01:35:12,440 DIALOGUES HOW TO BALANCE THOSE 2482 01:35:12,440 --> 01:35:14,000 CONFLICTING CHALLENGES IS REALLY 2483 01:35:14,000 --> 01:35:14,600 IMPORTANT. 2484 01:35:14,600 --> 01:35:18,320 WE'RE ON THE SAME TEAM. 2485 01:35:18,320 --> 01:35:23,160 >> WHAT A WONDERFUL MOMENT. 2486 01:35:23,160 --> 01:35:25,680 ADVOCATES PARTICIPATE IN THE 2487 01:35:25,680 --> 01:35:28,280 PROCESS OF CLARIFYING FDA 2488 01:35:28,280 --> 01:35:29,640 GUIDANCE WITH INDUSTRY PARTNERS 2489 01:35:29,640 --> 01:35:32,240 IN THE ROOM BECAUSE I THINK 2490 01:35:32,240 --> 01:35:35,440 THERE'S STILL SOME 2491 01:35:35,440 --> 01:35:36,400 MISUNDERSTANDING AND 2492 01:35:36,400 --> 01:35:38,400 MISINTERPRETATION OF GUIDANCE 2493 01:35:38,400 --> 01:35:39,600 THAT'S ON THE STREET. 2494 01:35:39,600 --> 01:35:43,200 AND I THINK ANY EFFORTS WE CAN 2495 01:35:43,200 --> 01:35:46,200 HELP FOSTER TO GET INDUSTRY 2496 01:35:46,200 --> 01:35:47,400 PARTNERS TOGETHER WITH THE FDA 2497 01:35:47,400 --> 01:35:52,000 AND MAKE SURE WE'RE ALL ON THE 2498 01:35:52,000 --> 01:35:54,040 SAME PAGE, VARIOUS CATEGORIES 2499 01:35:54,040 --> 01:35:55,640 LIKE PEDIATRIC RESEARCH THAT 2500 01:35:55,640 --> 01:35:57,600 WE'LL TALK ABOUT LATER, AND I 2501 01:35:57,600 --> 01:35:58,800 THINK IN WHICH THAT'S REALLY 2502 01:35:58,800 --> 01:35:59,240 IMPORTANT. 2503 01:35:59,240 --> 01:36:01,400 WE HAVE A QUESTION UP TOP. 2504 01:36:01,400 --> 01:36:05,920 >> MY NAME IS MARIA, WITH CURE 2505 01:36:05,920 --> 01:36:06,240 MLD. 2506 01:36:06,240 --> 01:36:08,440 MY QUESTION IS FOR P.J. NOW THAT 2507 01:36:08,440 --> 01:36:11,320 HE'S OPENED UP THAT PANDOR'S A 2508 01:36:11,320 --> 01:36:13,440 BOX OF NEWBORN SCREENING. 2509 01:36:13,440 --> 01:36:15,240 >> HI, MARIA. 2510 01:36:15,240 --> 01:36:15,960 >> HI, P.J. 2511 01:36:15,960 --> 01:36:18,400 ONE OF THE THINGS I THOUGHT 2512 01:36:18,400 --> 01:36:23,440 ABOUT IS GIVEN THE FACT THAT WE 2513 01:36:23,440 --> 01:36:29,040 CAN'T WAIT 30 YEARS FOR ACH AND 2514 01:36:29,040 --> 01:36:32,760 -- NO, ACH DNC TO GIVE APPROVAL 2515 01:36:32,760 --> 01:36:35,480 AND DO LOBBYING IN 50 STATES, DO 2516 01:36:35,480 --> 01:36:38,480 WE TAKE IT OUT OF THE HANDS OF 2517 01:36:38,480 --> 01:36:39,960 THE GOVERNMENT AND PUT IT IN THE 2518 01:36:39,960 --> 01:36:42,160 PATIENT'S HANDS IN TERMS OF 2519 01:36:42,160 --> 01:36:45,000 ASKING FOR HOLE GENOME 2520 01:36:45,000 --> 01:36:48,640 SEQUENCING AT BIRTH FOR 2521 01:36:48,640 --> 01:36:50,400 TREATABLE AND HERITABLE 2522 01:36:50,400 --> 01:36:51,320 DISEASES, EXACERBATING HEALTH 2523 01:36:51,320 --> 01:36:52,600 INEQUITIES IN THE SYSTEM BUT 2524 01:36:52,600 --> 01:36:54,000 THAT'S LIKELY BEGINNING TO 2525 01:36:54,000 --> 01:36:56,680 HAPPEN, WHERE FAMILIES WHO HAVE 2526 01:36:56,680 --> 01:36:58,200 RESOURCES WILL JUST DO WHOLE 2527 01:36:58,200 --> 01:36:59,640 GENOME SEQUENCING AT BIRTH AND 2528 01:36:59,640 --> 01:37:01,480 SAY, LISTEN, I'M NOT GOING TO 2529 01:37:01,480 --> 01:37:04,680 WAIT FOR NORTH DAKOTA TO DO 2530 01:37:04,680 --> 01:37:05,880 THIS, AND I'M GOING TO THROW 2531 01:37:05,880 --> 01:37:09,320 THAT OUT TO YOU AND THEN THE 2532 01:37:09,320 --> 01:37:12,200 SECOND PART IS AS YOU PIONEER 2533 01:37:12,200 --> 01:37:14,720 THIS NEW ERA OF GENE THERAPY, 2534 01:37:14,720 --> 01:37:19,840 THERE HAS TO BE A TWIN 2535 01:37:19,840 --> 01:37:21,160 REVOLUTION IN DIAGNOSTIC 2536 01:37:21,160 --> 01:37:22,360 MEDICINE WITH NEUROLOGISTS 2537 01:37:22,360 --> 01:37:24,360 LEADING THE WAY AND BASICALLY 2538 01:37:24,360 --> 01:37:25,320 GETTING PEDIATRICIANS TO STOP 2539 01:37:25,320 --> 01:37:28,800 THINKING I NEED TO WAIT FOR THE 2540 01:37:28,800 --> 01:37:33,000 KID TO GET SICK TO DIAGNOSE, AND 2541 01:37:33,000 --> 01:37:35,320 LET'S WAIT AND SIGH, AS OPPOSED 2542 01:37:35,320 --> 01:37:38,040 TO LET'S USE WHOLE GENOME 2543 01:37:38,040 --> 01:37:39,560 SEQUENCING THE MINUTE I HAVE A 2544 01:37:39,560 --> 01:37:42,840 KID IN THE ICU OR EVEN I SEE LOW 2545 01:37:42,840 --> 01:37:43,120 TONE. 2546 01:37:43,120 --> 01:37:46,320 I DON'T KNOW WHAT THE ANSWER IS 2547 01:37:46,320 --> 01:37:50,600 HERE BUT THERE HAS TO BE A TWIN 2548 01:37:50,600 --> 01:37:51,680 EDUCATIONAL EFFORT FOR FRONTLINE 2549 01:37:51,680 --> 01:37:53,760 PCP PEOPLE TO GET READY FOR GENE 2550 01:37:53,760 --> 01:37:55,080 THERAPY BECAUSE THEY ARE NOT 2551 01:37:55,080 --> 01:37:56,760 DIAGNOSING OUR KIDS IN TIME. 2552 01:37:56,760 --> 01:37:58,480 THAT'S MY QUESTION. 2553 01:37:58,480 --> 01:38:00,200 SORRY, P.J. 2554 01:38:00,200 --> 01:38:01,320 >> OH, SURE. 2555 01:38:01,320 --> 01:38:02,760 NO, IT'S A GREAT QUESTION MARIA, 2556 01:38:02,760 --> 01:38:04,440 AND YOU KNOW THIS ISSUE BETTER 2557 01:38:04,440 --> 01:38:06,160 THAN ANYBODY. 2558 01:38:06,160 --> 01:38:08,560 I THINK WE ARE FUNDING SOME WORK 2559 01:38:08,560 --> 01:38:12,160 AND THERE'S BEEN SOME PAPERS 2560 01:38:12,160 --> 01:38:14,120 PUBLISHED RECENTLY FROM WORK 2561 01:38:14,120 --> 01:38:17,320 FUNDED BY THE CTSA PROGRAM, 2562 01:38:17,320 --> 01:38:20,040 STEPHEN KINGSMORE AND 2563 01:38:20,040 --> 01:38:22,560 COLLEAGUES, SHOWING THE BENEFIT 2564 01:38:22,560 --> 01:38:24,200 OF WHOLE GENOME SEQUENCING FOR 2565 01:38:24,200 --> 01:38:27,160 PATIENTS IN THE ICU AND THEY 2566 01:38:27,160 --> 01:38:28,160 HAVE BEEN DEMONSTRATING THE 2567 01:38:28,160 --> 01:38:30,000 FINANCIAL IMPACT OF THAT, THAT'S 2568 01:38:30,000 --> 01:38:31,080 SOMETHING THAT'S QUITE 2569 01:38:31,080 --> 01:38:32,080 IMPORTANT. 2570 01:38:32,080 --> 01:38:36,640 I CAN PUT SOME LINKS IN THE CHAT 2571 01:38:36,640 --> 01:38:37,640 FOR THAT. 2572 01:38:37,640 --> 01:38:40,720 BUT I THINK THERE'S AN EVEN MUCH 2573 01:38:40,720 --> 01:38:43,040 BROADER PROBLEM WHICH IS WHAT 2574 01:38:43,040 --> 01:38:44,440 ABOUT FINDING KIDS WHO AREN'T 2575 01:38:44,440 --> 01:38:45,760 SYMPTOMATIC AT ALL YET BUT WILL 2576 01:38:45,760 --> 01:38:48,160 BE IN A COUPLE YEARS, AND I 2577 01:38:48,160 --> 01:38:52,000 THINK WE'VE ALL SEEN THE STORY 2578 01:38:52,000 --> 01:38:53,840 OF NILA AND THE DEVELOPMENT, IN 2579 01:38:53,840 --> 01:38:58,000 THE COURSE OF A YEAR, AND I LOOK 2580 01:38:58,000 --> 01:39:00,640 AT THAT AND THINK WHAT WOULD 2581 01:39:00,640 --> 01:39:02,080 HAVE HAPPENED IF THEY DIAGNOSED 2582 01:39:02,080 --> 01:39:04,960 NILA AS A NEWBORN. 2583 01:39:04,960 --> 01:39:06,680 SHE HAD NO SYMPTOMS AS A 2584 01:39:06,680 --> 01:39:07,240 NEWBORN. 2585 01:39:07,240 --> 01:39:12,040 IF WE DON'T HAVE A MUCH BROADER 2586 01:39:12,040 --> 01:39:14,160 NEWBORN SCREENING WE'RE GOING TO 2587 01:39:14,160 --> 01:39:15,880 PRIMARILY BE LOOKING AT PEOPLE 2588 01:39:15,880 --> 01:39:16,760 SYMPTOMATIC, THAT'S A 2589 01:39:16,760 --> 01:39:19,480 FUNDAMENTAL PROBLEM. 2590 01:39:19,480 --> 01:39:22,240 THE SOLUTION LIES BEYOND NIH, 2591 01:39:22,240 --> 01:39:23,520 BUT THAT'S WHY WE HAD THAT 2592 01:39:23,520 --> 01:39:25,520 SERIES OF MEETINGS AND I PUT THE 2593 01:39:25,520 --> 01:39:28,480 LINK IN THE CHAT TO RAISE THIS 2594 01:39:28,480 --> 01:39:29,680 ISSUE BECAUSE HOW YOU CHANGE 2595 01:39:29,680 --> 01:39:32,200 THAT, AS I SAID THAT GOES BEYOND 2596 01:39:32,200 --> 01:39:33,640 NIH TO A DIFFERENT PART OF THE 2597 01:39:33,640 --> 01:39:35,480 GOVERNMENT, I THINK THERE'S A 2598 01:39:35,480 --> 01:39:37,560 NEED FOR REAL CREATIVE SOLUTIONS 2599 01:39:37,560 --> 01:39:40,080 THERE, LET ME JUST PUT IT THAT 2600 01:39:40,080 --> 01:39:40,240 WAY. 2601 01:39:40,240 --> 01:39:44,800 >> I WAS VERY ENCOURAGED TO HEAR 2602 01:39:44,800 --> 01:39:49,360 ABOUT A PROGRAM THAT IS TRAINING 2603 01:39:49,360 --> 01:39:51,680 OB/GYNs IN ISSUES OF GENETIC 2604 01:39:51,680 --> 01:39:52,440 SCREENING. 2605 01:39:52,440 --> 01:39:55,480 THEY MADE THE POINT THAT 2606 01:39:55,480 --> 01:39:57,120 FREQUENTLY THROUGH THE IVF 2607 01:39:57,120 --> 01:39:59,360 PROCESS GENETIC SCREENING AND 2608 01:39:59,360 --> 01:40:00,720 COUNSELORS ARE BUILT INTO THE 2609 01:40:00,720 --> 01:40:06,440 PROCESS BUT NOBODY BUILDS INTO 2610 01:40:06,440 --> 01:40:09,080 THE NORMAL OBSTETRIC PROCESS. 2611 01:40:09,080 --> 01:40:10,600 HAVE THAT CONVERSATION WITH YOUR 2612 01:40:10,600 --> 01:40:11,720 OB PRIOR TO DELIVERY ABOUT 2613 01:40:11,720 --> 01:40:17,000 GENETIC SCREENING AND TESTING TO 2614 01:40:17,000 --> 01:40:18,200 HAVE GENETIC COUNSELORS AS PART 2615 01:40:18,200 --> 01:40:20,200 OF THE OB TEAM CAN GO A LONG WAY 2616 01:40:20,200 --> 01:40:23,800 TOWARDS THAT. 2617 01:40:23,800 --> 01:40:34,240 >> PENNY HAS A QUESTION. 2618 01:40:38,240 --> 01:40:39,120 >> ASSOCIATION FOR 2619 01:40:39,120 --> 01:40:42,080 FRONTOTEMPORAL DEGENERATION, 2620 01:40:42,080 --> 01:40:47,680 REPRESENTING ADULT SIDE. 2621 01:40:47,680 --> 01:40:53,280 SOME CAUSES ARE AUTOSOMAL 2622 01:40:53,280 --> 01:41:00,280 DOMINANT, ONE OF THE THINGS THT 2623 01:41:00,280 --> 01:41:02,360 WE PUT FORWARD IS WE DON'T 2624 01:41:02,360 --> 01:41:06,520 RECOMMEND GENETIC TESTING FOR 2625 01:41:06,520 --> 01:41:08,720 EVERYBODY BECAUSE THERE ARE 2626 01:41:08,720 --> 01:41:09,560 RAMIFICATIONS OF INSURANCE, 2627 01:41:09,560 --> 01:41:10,560 RAMIFICATIONS FOR JUST DO YOU 2628 01:41:10,560 --> 01:41:14,600 WANT TO KNOW WHAT'S IN YOUR 2629 01:41:14,600 --> 01:41:14,840 FUTURE? 2630 01:41:14,840 --> 01:41:24,800 BUT AT THE SAME TIME WE HAVE 2631 01:41:24,800 --> 01:41:30,720 HARD ACCESS TO GENETIC 2632 01:41:30,720 --> 01:41:32,800 COUNSELORS, YOU GOT DIAGNOSED 2633 01:41:32,800 --> 01:41:36,080 BUT IT MAY NOT BE CAUSED BY A 2634 01:41:36,080 --> 01:41:37,520 GENETIC VARIANT. 2635 01:41:37,520 --> 01:41:43,200 MOST PEOPLE CAN'T ACCESS THE 2636 01:41:43,200 --> 01:41:44,520 COUNSELORS, USUALLY WITHOUT 2637 01:41:44,520 --> 01:41:53,200 EXPERTISE IN THAT FORM OF 2638 01:41:53,200 --> 01:41:53,840 NEURODEGENERATION, SIMILARITIES 2639 01:41:53,840 --> 01:41:55,800 AND DIFFERENCES, THE EARLIER WE 2640 01:41:55,800 --> 01:41:58,760 STOP, THE EARLIER WE TREAT, THE 2641 01:41:58,760 --> 01:41:59,840 BETTER WE'LL BE ABLE TO HELP 2642 01:41:59,840 --> 01:42:01,640 PEOPLE BEFORE THE BRAIN HAS 2643 01:42:01,640 --> 01:42:04,800 GOTTEN DAMAGED, BUT THERE'S SOME 2644 01:42:04,800 --> 01:42:06,520 PROFOUND DIFFERENCES IN TERMS OF 2645 01:42:06,520 --> 01:42:08,600 WHO GETS TO DECIDE IF AND WHEN 2646 01:42:08,600 --> 01:42:11,120 YOU GO THROUGH THAT TESTING, AND 2647 01:42:11,120 --> 01:42:12,320 WHAT ARE THE RESOURCES THAT ARE 2648 01:42:12,320 --> 01:42:16,360 OUT THERE TO HELP THE PEOPLE AND 2649 01:42:16,360 --> 01:42:17,800 FAMILIES MAKE INFORMED DECISIONS 2650 01:42:17,800 --> 01:42:18,760 FOR THEMSELVES? 2651 01:42:18,760 --> 01:42:20,560 JUST A GENERAL COMMENT FOR 2652 01:42:20,560 --> 01:42:30,440 ANYONE TO RESPOND TO. 2653 01:42:30,440 --> 01:42:31,240 >> WELL SAID. 2654 01:42:31,240 --> 01:42:33,560 I THINK IT'S CERTAINLY A 2655 01:42:33,560 --> 01:42:38,480 CHALLENGE WITHIN THAT 2656 01:42:38,480 --> 01:42:38,760 POPULATION. 2657 01:42:38,760 --> 01:42:40,880 ONE OF THE BIGGEST PROBLEMS, THE 2658 01:42:40,880 --> 01:42:42,440 NEUROLOGISTS ARE NOT OFFERING 2659 01:42:42,440 --> 01:42:43,520 WITH CLEAR EXPLANATION HOW TO 2660 01:42:43,520 --> 01:42:48,920 THINK ABOUT IT. 2661 01:42:48,920 --> 01:42:52,320 2662 01:42:52,320 --> 01:42:55,800 PARKINSON'S DISEASE, 2663 01:42:55,800 --> 01:42:56,680 FRONTOTEMPORAL DEMENTIA, WITH 2664 01:42:56,680 --> 01:42:57,280 DISEASE-MODIFYING THINGS OUT 2665 01:42:57,280 --> 01:42:58,000 THERE, PATIENTS SHOULD BE MADE 2666 01:42:58,000 --> 01:43:03,120 AWARE IF YOU ARE HAVE A GENETIC 2667 01:43:03,120 --> 01:43:05,320 SUBTYPE THERE MAY BE A CLINICAL 2668 01:43:05,320 --> 01:43:06,520 TRIAL OR EVENTUALLY A THERAPY 2669 01:43:06,520 --> 01:43:07,400 YOU CAN TAKE. 2670 01:43:07,400 --> 01:43:08,840 BUT THE ONLY WAY TO KNOW IF IT'S 2671 01:43:08,840 --> 01:43:13,520 RIGHT FOR YOU IF YOU GET GENETIC 2672 01:43:13,520 --> 01:43:15,720 TESTING AND EXPLAIN WHAT THAT 2673 01:43:15,720 --> 01:43:16,080 ENTAILS. 2674 01:43:16,080 --> 01:43:19,880 EACH STATE HAS THEIR OWN 2675 01:43:19,880 --> 01:43:20,160 CHALLENGES. 2676 01:43:20,160 --> 01:43:23,400 EACH STATE HAS DIFFERENT 2677 01:43:23,400 --> 01:43:24,360 CHALLENGES, EXPERT CENTERS 2678 01:43:24,360 --> 01:43:26,360 AVAILABLE, WHERE THESE PEOPLE 2679 01:43:26,360 --> 01:43:27,160 ARE BEING DIAGNOSED. 2680 01:43:27,160 --> 01:43:29,760 I THINK THAT'S THE FIRST HURDLE, 2681 01:43:29,760 --> 01:43:32,600 JUST FOR THE CLINICIAN TO BE 2682 01:43:32,600 --> 01:43:34,000 AWARE THAT THESE POTENTIAL 2683 01:43:34,000 --> 01:43:36,200 THERAPIES ARE OUT THERE, THAT 2684 01:43:36,200 --> 01:43:37,640 EVEN JUST THE GENETIC UNDERLYING 2685 01:43:37,640 --> 01:43:42,880 OF THE DISEASE IS A REAL 2686 01:43:42,880 --> 01:43:43,760 POSSIBILITY. 2687 01:43:43,760 --> 01:43:46,200 AND THEN EXPLAINING IT IN A 2688 01:43:46,200 --> 01:43:48,480 CLEAR WAY TO THE PATIENT TO MAKE 2689 01:43:48,480 --> 01:43:51,120 THEIR INFORMED DECISION, I 2690 01:43:51,120 --> 01:43:52,640 TOTALLY AGREE CLINICIANS DON'T 2691 01:43:52,640 --> 01:43:54,280 HAVE TIME OR EXPERTISE TO DO THE 2692 01:43:54,280 --> 01:43:57,000 COUNSELING IN THE WHICH YOU WANT 2693 01:43:57,000 --> 01:43:58,120 IT DONE. 2694 01:43:58,120 --> 01:44:01,800 SO WE REALLY -- THOSE EXPERT 2695 01:44:01,800 --> 01:44:03,160 CENTERS WITH GENETIC COUNSELORS 2696 01:44:03,160 --> 01:44:05,120 ARE AN AMAZING RESOURCE BUT 2697 01:44:05,120 --> 01:44:06,080 HAVING ACCESS TO COUNSELING IN 2698 01:44:06,080 --> 01:44:11,800 GENERAL IS REALLY IMPORTANT. 2699 01:44:11,800 --> 01:44:12,560 THERE'S REALLY INTERESTING 2700 01:44:12,560 --> 01:44:17,680 SCREENING PROGRAMS OUT THERE IN 2701 01:44:17,680 --> 01:44:21,720 THE PARKINSON'S DISEASE, THE 2702 01:44:21,720 --> 01:44:22,600 PARKINSON'S FOUNDATION SPONSORS 2703 01:44:22,600 --> 01:44:24,480 INCLUDING GENETIC TESTING AND 2704 01:44:24,480 --> 01:44:25,360 COUNSELING, VIRTUALLY AS WELL, 2705 01:44:25,360 --> 01:44:29,200 THAT HAS TO BE BUILT IN AND I 2706 01:44:29,200 --> 01:44:32,360 TOTALLY AGREE. 2707 01:44:32,360 --> 01:44:33,920 >> ARE THERE ANY FINAL QUESTIONS 2708 01:44:33,920 --> 01:44:35,560 FROM ANYONE IN THE ROOM OR 2709 01:44:35,560 --> 01:44:39,480 ONLINE? 2710 01:44:39,480 --> 01:44:40,800 HEARING NO TAKERS, WE CAN GIVE 2711 01:44:40,800 --> 01:44:43,200 YOU BACK A LITTLE BIT OF YOUR 2712 01:44:43,200 --> 01:44:45,920 MORNING TO PREPARE FOR LUNCH, 2713 01:44:45,920 --> 01:44:50,080 AND I WOULD LIKE YOU TO JOIN ME 2714 01:44:50,080 --> 01:44:52,640 IN THANKING THIS TERRIFIC PANEL. 2715 01:44:52,640 --> 01:45:01,920 [APPLAUSE] 2716 01:45:01,920 --> 01:45:08,960 >> WE'LL TURN IT OVER TO JILL 2717 01:45:08,960 --> 01:45:10,640 MORRIS, PROGRAM DIRECTOR, 2718 01:45:10,640 --> 01:45:12,720 DIVISION OF NEUROSCIENCE. 2719 01:45:12,720 --> 01:45:13,920 >> HI, EVERYONE. 2720 01:45:13,920 --> 01:45:16,240 TODAY WE'LL TALK ABOUT THE 2721 01:45:16,240 --> 01:45:17,560 ETHICS AND CHALLENGES IN GENE 2722 01:45:17,560 --> 01:45:20,840 THERAPY, I THINK THIS WILL BE A 2723 01:45:20,840 --> 01:45:22,920 VERY LIVELY DISCUSSION. 2724 01:45:22,920 --> 01:45:26,960 SO, I AM AN NINDS PROGRAM 2725 01:45:26,960 --> 01:45:29,480 DIRECTOR, I DID MY THESIS WORK 2726 01:45:29,480 --> 01:45:32,800 IN A GENE THERAPY LAB BACK IN 2727 01:45:32,800 --> 01:45:34,280 THE EARLY 1990s. 2728 01:45:34,280 --> 01:45:36,720 I WAS TELLING MARIA, I TRAINED 2729 01:45:36,720 --> 01:45:40,240 WITH PAUL ORCHARD, WHO DOES MANY 2730 01:45:40,240 --> 01:45:45,360 BONE MARROW TRANSPLANTS FOR GENE 2731 01:45:45,360 --> 01:45:45,560 THERAPY. 2732 01:45:45,560 --> 01:45:47,200 I'M RESPONSIBLE FOR MULTI-RARE 2733 01:45:47,200 --> 01:45:48,560 AND ULTRA RARE NEUROLOGICAL 2734 01:45:48,560 --> 01:45:55,080 DISORDERS THAT ARE CURRENTLY 2735 01:45:55,080 --> 01:45:57,880 USING GENE THERAPY. 2736 01:45:57,880 --> 01:46:03,200 I'M A MEMBER OF THE BESPOKE TEAM 2737 01:46:03,200 --> 01:46:06,160 AND ALSO NINDS LEAD FOR THE RARE 2738 01:46:06,160 --> 01:46:09,080 DISEASE CLINICAL RESEARCH 2739 01:46:09,080 --> 01:46:11,720 NETWORK FOR THE RDCRN THROUGH 2740 01:46:11,720 --> 01:46:12,120 NCATS. 2741 01:46:12,120 --> 01:46:13,080 WHICH ALLOWS TO ESTABLISH 2742 01:46:13,080 --> 01:46:14,720 CLINICAL TRIAL READINESS FOR 2743 01:46:14,720 --> 01:46:18,240 RARE AND ULTRA RARE DISEASES, 2744 01:46:18,240 --> 01:46:20,360 AND NINDS SUPPORTS 11 OF THE 20 2745 01:46:20,360 --> 01:46:22,480 CONSORTIUM THAT ARE FUNDED 2746 01:46:22,480 --> 01:46:24,120 THROUGH THE RDCRN. 2747 01:46:24,120 --> 01:46:25,640 WE'RE GOING TO BEGIN, I'LL HAVE 2748 01:46:25,640 --> 01:46:27,880 EACH OF THE SPEAKERS INTRODUCE 2749 01:46:27,880 --> 01:46:30,160 THEMSELVES, AND THEN I WANT THEM 2750 01:46:30,160 --> 01:46:33,160 TO ANSWER THIS ONE QUESTION, 2751 01:46:33,160 --> 01:46:33,840 DURING THEIR INTRODUCTION, WHAT 2752 01:46:33,840 --> 01:46:37,080 DO YOU THINK ARE THE CURRENT 2753 01:46:37,080 --> 01:46:38,480 SIGNIFICANT CHALLENGES AND 2754 01:46:38,480 --> 01:46:40,200 ETHICAL CONCERNS FOR GENE 2755 01:46:40,200 --> 01:46:41,200 THERAPY? 2756 01:46:41,200 --> 01:46:44,800 WHY DON'T WE START WITH REGHAN. 2757 01:46:44,800 --> 01:46:47,960 >> THANK YOU, I'M REGHAN FOLEY, 2758 01:46:47,960 --> 01:46:48,960 PEDIATRIC NEUROLOGIST, 2759 01:46:48,960 --> 01:46:49,840 NEUROMUSCULAR SPECIALIST, SENIOR 2760 01:46:49,840 --> 01:46:54,440 RESEARCH PHYSICIAN AT THE NINDS. 2761 01:46:54,440 --> 01:46:57,920 A LOT OF OUR WORK IS NATURAL 2762 01:46:57,920 --> 01:47:01,800 HISTORY WORK AND CLINICAL TRIAL 2763 01:47:01,800 --> 01:47:02,600 READINESS, LOOKING FOR 2764 01:47:02,600 --> 01:47:03,080 BIOMARKERS. 2765 01:47:03,080 --> 01:47:04,400 WE'VE HAD A FEW GENE THERAPY 2766 01:47:04,400 --> 01:47:09,080 STUDIES, MANY IN THE PIPELINE SO 2767 01:47:09,080 --> 01:47:15,960 WE DID AN INTRATHECAL AAV9 AND 2768 01:47:15,960 --> 01:47:26,480 SYSTEMIC WITH AAV8, THEY HAVE 2769 01:47:26,920 --> 01:47:28,720 BEEN VERY EDUCATIONAL 2770 01:47:28,720 --> 01:47:29,200 EXPERIENCES. 2771 01:47:29,200 --> 01:47:31,400 FOR ME, ETHICAL CONCERNS IN THE 2772 01:47:31,400 --> 01:47:33,200 GENE THERAPY FIELD, THERE IS 2773 01:47:33,200 --> 01:47:36,800 REASON FOR OPTIMISM, ALSO REASON 2774 01:47:36,800 --> 01:47:41,040 TO STRETCH AND HOLD THE FIELD TO 2775 01:47:41,040 --> 01:47:43,360 A HIGHER STANDARD OF 2776 01:47:43,360 --> 01:47:45,520 TRANSPARENCY BETWEEN THE 2777 01:47:45,520 --> 01:47:46,760 SPONSOR, ACADEMIC OR INDUSTRY, 2778 01:47:46,760 --> 01:47:47,640 TRANSPARENCY IS ABOUT THE 2779 01:47:47,640 --> 01:47:49,920 CONSTRUCT, ABOUT THE GENE 2780 01:47:49,920 --> 01:47:51,560 THERAPY ITSELF. 2781 01:47:51,560 --> 01:47:53,400 MAKING SURE INVESTIGATORS ARE 2782 01:47:53,400 --> 01:47:54,200 AWARE PRE-CLINICAL DATA THINGS 2783 01:47:54,200 --> 01:47:59,680 TO LOOK OUT FOR TRANSPARENCY, N 2784 01:47:59,680 --> 01:48:03,400 REAL TIME AS ADVERSE EVENTS 2785 01:48:03,400 --> 01:48:04,280 BECOME APPARENT. 2786 01:48:04,280 --> 01:48:06,240 IT'S IMPERATIVE TO BE AWARE OF 2787 01:48:06,240 --> 01:48:07,360 WHAT'S GOING ON AT OTHER SITES 2788 01:48:07,360 --> 01:48:08,560 IN REAL TEAM. 2789 01:48:08,560 --> 01:48:10,200 IF THAT DOESN'T HAPPEN WE CAN'T 2790 01:48:10,200 --> 01:48:11,960 OPTIMIZE THE SAFETY FOR THE 2791 01:48:11,960 --> 01:48:14,120 PATIENTS ENROLLED AS WELL AS 2792 01:48:14,120 --> 01:48:17,440 MITIGATE RISK FOR FUTURE 2793 01:48:17,440 --> 01:48:17,680 PATIENTS. 2794 01:48:17,680 --> 01:48:20,120 BUT ALSO THE WORD COMES TO MIND 2795 01:48:20,120 --> 01:48:20,480 RESPONSIBILITY. 2796 01:48:20,480 --> 01:48:22,760 AS A FIELD TO MAKE GENE THERAPY 2797 01:48:22,760 --> 01:48:24,120 MORE AVAILABLE BUT ALSO 2798 01:48:24,120 --> 01:48:24,960 RESPONSIBILITY TO BE RESPONSIVE 2799 01:48:24,960 --> 01:48:27,800 TO THE PATIENTS AND FAMILIES. 2800 01:48:27,800 --> 01:48:28,800 SO, THAT RESPONSIVENESS, WHAT'S 2801 01:48:28,800 --> 01:48:30,440 NECESSARY AND IMPORTANT FOR 2802 01:48:30,440 --> 01:48:31,880 OUTCOME MEASURES, BUT ALSO 2803 01:48:31,880 --> 01:48:33,320 RECOGNIZING PATIENTS AND THEIR 2804 01:48:33,320 --> 01:48:34,600 FAMILIES ARE THE TRUE EXPERTS, 2805 01:48:34,600 --> 01:48:41,520 THEY NEVER CHOSE TO BE EXPERTS 2806 01:48:41,520 --> 01:48:42,800 IN RARE DISEASES, WE SHOWS TO BE 2807 01:48:42,800 --> 01:48:44,800 EXPERTS BUT THEY LIVE THEY. 2808 01:48:44,800 --> 01:48:47,400 LIVED EXPERIENCE HAS TO BE 2809 01:48:47,400 --> 01:48:51,360 RESPONDED TO, WE HAVE TO BE 2810 01:48:51,360 --> 01:48:53,320 RESPONSIBLE TO FAMILIES WHILE 2811 01:48:53,320 --> 01:48:55,720 WE'RE LAUNCHING AND DURING AND 2812 01:48:55,720 --> 01:48:57,480 ADVERSE EVENTS, WE HAVE 2813 01:48:57,480 --> 01:48:59,560 RESPONSIBILITY TO QUICKLY AND 2814 01:48:59,560 --> 01:49:03,840 TRANSPARENTLY REPORT EXACTLY 2815 01:49:03,840 --> 01:49:04,800 WHAT HAPPENED. 2816 01:49:04,800 --> 01:49:13,120 EVERYONE NEEDS TO KNOW EXACTLY 2817 01:49:13,120 --> 01:49:14,040 WHAT HAPPENED. 2818 01:49:14,040 --> 01:49:22,520 >> I'M A PATIENT ADVOCATE FOR 2819 01:49:22,520 --> 01:49:33,000 GENE THERAPY, AN ACCIDENTAL 2820 01:49:39,120 --> 01:49:39,320 ADVOCATE. 2821 01:49:39,320 --> 01:49:43,560 SINCE I WAS 12 MY YOUNGEST 2822 01:49:43,560 --> 01:49:45,040 DAUGHTER WAS DIAGNOSED WITH 2823 01:49:45,040 --> 01:49:46,600 LEUKODYSTROPHY, WE WERE AT THE 2824 01:49:46,600 --> 01:49:47,160 CHILDREN'S HOSPITAL AT 2825 01:49:47,160 --> 01:49:48,080 PHILADELPHIA WITH ONE OF THE 2826 01:49:48,080 --> 01:49:51,200 BEST NEUROLOGY TEAMS IN THE 2827 01:49:51,200 --> 01:49:57,000 WORLD, AND ON THE FIFTH JULY A 2828 01:49:57,000 --> 01:50:04,320 YOUNG DOCTOR TOLD MY MY DAUGHTER 2829 01:50:04,320 --> 01:50:06,520 HAD MLD, BASICALLY ALS AND 2830 01:50:06,520 --> 01:50:10,440 ALZHEIMER'S IN A 2-YEAR-OLD. 2831 01:50:10,440 --> 01:50:12,680 THEY PRESENT AT BIRTH COMPLETELY 2832 01:50:12,680 --> 01:50:15,520 NORMAL AND HEALTHY. 2833 01:50:15,520 --> 01:50:19,360 MY DAUGHTER HAD INHERITED THE 2834 01:50:19,360 --> 01:50:21,320 DISORDER, INHERITED TWO FAULTY 2835 01:50:21,320 --> 01:50:23,040 COPIES OF THE GENE WHICH MADE IT 2836 01:50:23,040 --> 01:50:24,720 IMPOSSIBLE FOR HER BODY TO PRO 2837 01:50:24,720 --> 01:50:27,080 PRODUCE A SINGLE PROTEIN. 2838 01:50:27,080 --> 01:50:33,120 THE DEFICIENCY OF THE PROTEIN 2839 01:50:33,120 --> 01:50:36,680 WAS CAUSING SULFOTIDES TO 2840 01:50:36,680 --> 01:50:37,760 ACCUMULATE IN HER BRAIN, 2841 01:50:37,760 --> 01:50:38,920 BASICALLY KILLING TEAR SLOWLY. 2842 01:50:38,920 --> 01:50:41,240 AS A YOUNG DOCTOR WHO JUST 2843 01:50:41,240 --> 01:50:42,240 FINISHED RESIDENCY AND 2844 01:50:42,240 --> 01:50:47,800 FELLOWSHIP EXPLAINED TO ME ABOUT 2845 01:50:47,800 --> 01:50:50,520 MLD, AND MY HUSBAND HELD OUR 2846 01:50:50,520 --> 01:50:53,600 LITTLE GIRL IN HIS ARMS I HADN'T 2847 01:50:53,600 --> 01:50:55,920 SPOKEN IN ABOUT A WEEK SINCE WE 2848 01:50:55,920 --> 01:51:01,240 HAD FIRST GONE TO THE HOSPITAL 2849 01:51:01,240 --> 01:51:10,480 AND WE WAS DIAGNOSED, 5th JULY 2850 01:51:10,480 --> 01:51:11,160 DIAGNOSIS CONFIRMED, HADN'T 2851 01:51:11,160 --> 01:51:12,720 SPOKEN IN A WEEK AND THEN 2852 01:51:12,720 --> 01:51:13,840 COULDN'T SHUT UP. 2853 01:51:13,840 --> 01:51:15,160 I UNDERSTAND MY DAUGHTER IS 2854 01:51:15,160 --> 01:51:15,920 DYING. 2855 01:51:15,920 --> 01:51:17,040 SHE SHOWED ME THE MRI, THE 2856 01:51:17,040 --> 01:51:19,960 DAMAGE TO THE BRAIN WAS SO 2857 01:51:19,960 --> 01:51:21,400 CATASTROPHIC, YOU COULD SEE THE 2858 01:51:21,400 --> 01:51:24,920 BRAIN RECEDING FROM THE SKULL. 2859 01:51:24,920 --> 01:51:27,000 AND I UNDERSTOOD MY DAUGHTER 2860 01:51:27,000 --> 01:51:28,600 WOULD NEVER BE SAVED, A DEAD 2861 01:51:28,600 --> 01:51:30,480 NEURON IS A DEAD NEURON. 2862 01:51:30,480 --> 01:51:32,440 BUT I SAID TO HER, WILL YOU EVER 2863 01:51:32,440 --> 01:51:34,520 HAVE A TREATMENT? 2864 01:51:34,520 --> 01:51:35,720 SHE SAID YES. 2865 01:51:35,720 --> 01:51:37,360 THERE'S A THING CALLED GENE 2866 01:51:37,360 --> 01:51:37,800 THERAPY. 2867 01:51:37,800 --> 01:51:40,000 I SAID TO DR. WALDMANN, I DON'T 2868 01:51:40,000 --> 01:51:42,880 KNOW WHAT EVER POSSESSED ME TO 2869 01:51:42,880 --> 01:51:44,800 SAY THIS, I SAID, WHEN YOU FIND 2870 01:51:44,800 --> 01:51:46,320 THIS GENE THERAPY, WHEN YOU MAKE 2871 01:51:46,320 --> 01:51:48,400 IT WORK, I WANT YOU TO FIND ME. 2872 01:51:48,400 --> 01:51:50,160 I DON'T KNOW HOW OLD I'LL BE. 2873 01:51:50,160 --> 01:51:51,720 I DON'T KNOW WHERE I WILL BE. 2874 01:51:51,720 --> 01:51:53,240 BUT I WANT YOU TO FIND ME AND 2875 01:51:53,240 --> 01:51:56,080 GET ME ON A PLANE TO ICELAND OR 2876 01:51:56,080 --> 01:51:58,040 PARIS OR WHEREVER YOU FIND THIS 2877 01:51:58,040 --> 01:52:01,000 BABY, THAT YOU CAN GIVE GENE 2878 01:52:01,000 --> 01:52:02,560 THERAPY TO, AND I WANT TO MEET 2879 01:52:02,560 --> 01:52:04,080 HER AND HER MOTHER. 2880 01:52:04,080 --> 01:52:05,840 IN I'M REALLY LUCKY, MAY BE 2881 01:52:05,840 --> 01:52:07,920 SHE'LL HAVE BROWN EYES AND CURLY 2882 01:52:07,920 --> 01:52:12,640 BROWN HAIR LIKE MY LITTLE GIRL. 2883 01:52:12,640 --> 01:52:15,160 AND DR. WALDMAN HUGGED ME AND 2884 01:52:15,160 --> 01:52:16,920 SAID I'M SO SORRY. 2885 01:52:16,920 --> 01:52:19,640 A YEAR LATER, THIS IS ABSOLUTELY 2886 01:52:19,640 --> 01:52:22,240 TRUE, YOU CAN CHECK, A YEAR 2887 01:52:22,240 --> 01:52:25,960 LATER A YOUNG RESEARCHER NAMED 2888 01:52:25,960 --> 01:52:27,520 AL SANDRA BIFFI PUBLISHED IN THE 2889 01:52:27,520 --> 01:52:30,480 JOURNAL OF SCIENCE, SHE TREATED 2890 01:52:30,480 --> 01:52:32,800 SUCCESSFULLY THREE CHILDREN WITH 2891 01:52:32,800 --> 01:52:33,640 THIS. 2892 01:52:33,640 --> 01:52:36,520 I CALLED DR. WALDMANN, I WAS NOT 2893 01:52:36,520 --> 01:52:37,040 HAPPY. 2894 01:52:37,040 --> 01:52:39,360 I WAS PRETTY PISSED OFF. 2895 01:52:39,360 --> 01:52:41,840 I SAID, AMY, WHAT THE HECK? 2896 01:52:41,840 --> 01:52:47,160 YOU SAID 10, 20 YEARS. 2897 01:52:47,160 --> 01:52:49,240 ONE YEAR AFTER CAL'S DIAGNOSIS? 2898 01:52:49,240 --> 01:52:51,960 MARIA, THERE WAS NEVER ANY WAY 2899 01:52:51,960 --> 01:53:00,360 TO SAVE CAL. 2900 01:53:00,360 --> 01:53:04,200 WE'VE NEVER SAVED, BY THE TIME 2901 01:53:04,200 --> 01:53:05,640 THEY SHOW SYMPTOMS IT IS OUT OF 2902 01:53:05,640 --> 01:53:05,960 CONTROL. 2903 01:53:05,960 --> 01:53:08,040 YOU NEVER COULD HAVE SAVED CAL. 2904 01:53:08,040 --> 01:53:11,800 THESE FAMILIES IN DR. BIFFI'S 2905 01:53:11,800 --> 01:53:14,640 TRIAL PAID AN INCREDIBLE PRICE, 2906 01:53:14,640 --> 01:53:15,960 SACRIFICED ONE CHILD TO THE 2907 01:53:15,960 --> 01:53:17,480 DISEASE TO SAVE THE YOUNGER ONE. 2908 01:53:17,480 --> 01:53:19,600 SHE SAID YOU MUST HELP THEM. 2909 01:53:19,600 --> 01:53:22,400 I WAS STARTING A FOUNDATION, 2910 01:53:22,400 --> 01:53:23,320 SELLING CUPCAKES. 2911 01:53:23,320 --> 01:53:24,720 I DON'T KNOW WHY. 2912 01:53:24,720 --> 01:53:25,800 I NEEDED TO DO SOMETHING. 2913 01:53:25,800 --> 01:53:28,800 I WAS GOING TO GIVE THE MONEY TO 2914 01:53:28,800 --> 01:53:30,320 PALLIATIVE CARE, TO CHOP. 2915 01:53:30,320 --> 01:53:31,320 A LITTLE-KNOWN FACT. 2916 01:53:31,320 --> 01:53:34,080 I WAS RAISING MONEY FOR 2917 01:53:34,080 --> 01:53:34,720 PALLIATIVE CARE BECAUSE THAT'S 2918 01:53:34,720 --> 01:53:37,800 ALL THEY COULD DO FOR MY 2919 01:53:37,800 --> 01:53:38,040 DAUGHTER. 2920 01:53:38,040 --> 01:53:39,480 DR. WALDMAN SAID GIVE IT TO THE 2921 01:53:39,480 --> 01:53:44,040 GENE THERAPY FAMILIES, GET THEM 2922 01:53:44,040 --> 01:53:44,840 TO ITALY. 2923 01:53:44,840 --> 01:53:47,120 AND A YEAR LATER, THE FIRST 2924 01:53:47,120 --> 01:53:52,360 AMERICAN CHILD NEEDED TO GET TO 2925 01:53:52,360 --> 01:53:53,240 ITALY. 2926 01:53:53,240 --> 01:53:54,880 2014. 2927 01:53:54,880 --> 01:53:56,160 AND FOUND THEM ON FACEBOOK, AND 2928 01:53:56,160 --> 01:53:57,080 THEY FOUND ME. 2929 01:53:57,080 --> 01:53:58,720 FOR THE LAST TEN YEARS THAT'S 2930 01:53:58,720 --> 01:54:02,400 WHAT I HAVE DONE. 2931 01:54:02,400 --> 01:54:03,880 I HAVE SENT 27 CHILDREN TO 2932 01:54:03,880 --> 01:54:06,040 TREATMENT IN ITALY AND NOW IN 2933 01:54:06,040 --> 01:54:09,000 THE UNITED STATES WITH THE 2934 01:54:09,000 --> 01:54:11,640 AMAZING PAUL ORCHARD. 2935 01:54:11,640 --> 01:54:12,960 AND MY DREAM WHEN MY DAUGHTER 2936 01:54:12,960 --> 01:54:15,120 WAS GOING TO DIE HAS COME TRUE. 2937 01:54:15,120 --> 01:54:17,360 I CAN NOW SIT IN A ROOM FULL OF 2938 01:54:17,360 --> 01:54:21,960 CHILDREN WHO CAN SING AND DANCE 2939 01:54:21,960 --> 01:54:23,880 AND HAVE NO AWARENESS OF THE 2940 01:54:23,880 --> 01:54:26,960 FACT THAT THEY HAVE MLD. 2941 01:54:26,960 --> 01:54:27,480 MY DAUGHTER GRANTED ME A 2942 01:54:27,480 --> 01:54:29,800 FRONT-ROW SEAT TO THE BIGGEST 2943 01:54:29,800 --> 01:54:34,840 MEDICAL BREAKTHROUGH IN A 2944 01:54:34,840 --> 01:54:35,120 GENERATION. 2945 01:54:35,120 --> 01:54:38,680 SO, I NOW WORK WITH A COMPANY 2946 01:54:38,680 --> 01:54:39,360 CALLED ORCHARD THERAPEUTICS. 2947 01:54:39,360 --> 01:54:44,360 IN THE NEXT YEAR I CAN TELL YOU 2948 01:54:44,360 --> 01:54:46,680 WITH CERTAINTY THAT THAT GENE 2949 01:54:46,680 --> 01:54:48,560 THERAPY WILL BECOME A 2950 01:54:48,560 --> 01:54:49,600 COMMERCIALLY AVAILABLE THERAPY 2951 01:54:49,600 --> 01:54:50,720 IN THE UNITED STATES. 2952 01:54:50,720 --> 01:54:52,000 WE'RE NOW WORKING TO MAKE SURE 2953 01:54:52,000 --> 01:54:53,120 THAT EVERY CHILD IN THE UNITED 2954 01:54:53,120 --> 01:54:56,960 STATES IS SCREENED FOR THIS 2955 01:54:56,960 --> 01:55:03,520 DISORDER. 2956 01:55:03,520 --> 01:55:05,440 THIS HAS BEEN AN AMAZING JOURNEY 2957 01:55:05,440 --> 01:55:09,400 TO BE IN THE FIRST GENERATION OF 2958 01:55:09,400 --> 01:55:10,120 GENE THERAPIES. 2959 01:55:10,120 --> 01:55:12,080 IT IS SAFE. 2960 01:55:12,080 --> 01:55:13,360 IT IS DURABLE. 2961 01:55:13,360 --> 01:55:15,040 IT GETS ME TO THE CHALLENGE. 2962 01:55:15,040 --> 01:55:17,000 I'M SORRY I'M GOING A LITTLE 2963 01:55:17,000 --> 01:55:17,200 LONG. 2964 01:55:17,200 --> 01:55:18,640 IT'S A LONG STORY. 2965 01:55:18,640 --> 01:55:20,200 I DON'T HAVE ANY SCIENTIFIC 2966 01:55:20,200 --> 01:55:22,480 CREDENTIALS, MY Ph.D. IS IN 2967 01:55:22,480 --> 01:55:25,000 SOCIOLOGY, MY FATHER WOULD SAY 2968 01:55:25,000 --> 01:55:26,040 YOU FLUNKED CHEMISTRY TWICE, THE 2969 01:55:26,040 --> 01:55:28,720 FACT THAT YOU'RE AT THE NIH IS A 2970 01:55:28,720 --> 01:55:30,040 MIRACLE IN ITSELF. 2971 01:55:30,040 --> 01:55:32,120 THAT ANYONE IS LISTENING TO 2972 01:55:32,120 --> 01:55:35,520 ANYTHING YOU HAVE TO SAY GIVEN 2973 01:55:35,520 --> 01:55:38,840 YOU COULD NOT MASTER CHEMISTRY 2974 01:55:38,840 --> 01:55:40,400 IS AMUSING. 2975 01:55:40,400 --> 01:55:42,000 I PROBABLY HAVE MET MORE GENE 2976 01:55:42,000 --> 01:55:44,880 THERAPY PATIENTS THAN NEARLY 2977 01:55:44,880 --> 01:55:46,360 ANYONE IN THE WORLD. 2978 01:55:46,360 --> 01:55:48,480 THE FIRST CHALLENGE IS MONEY. 2979 01:55:48,480 --> 01:55:52,000 IN 2018 I PUBLISHED A PIECE IN 2980 01:55:52,000 --> 01:55:54,800 STAT I SAID THE SCIENCE OF GENE 2981 01:55:54,800 --> 01:55:57,400 THERAPY IS NOT AS HARD AS THE 2982 01:55:57,400 --> 01:55:59,080 MATH OF PAYING FOR IT. 2983 01:55:59,080 --> 01:56:01,400 AND WE WILL FACE SERIOUS 2984 01:56:01,400 --> 01:56:05,440 PROBLEMS IN TERMS OF 2985 01:56:05,440 --> 01:56:05,760 REIMBURSEMENT. 2986 01:56:05,760 --> 01:56:10,280 I HAD THE GREAT HONOR OF HELPING 2987 01:56:10,280 --> 01:56:12,200 GET APPROVAL IN GERMANY AT THE 2988 01:56:12,200 --> 01:56:15,320 NHS, THAT WAS EASY COMPARED TO 2989 01:56:15,320 --> 01:56:17,080 WHAT WE'LL BE FACING HERE. 2990 01:56:17,080 --> 01:56:18,560 IT'S A VERY SOLVABLE PROBLEM BUT 2991 01:56:18,560 --> 01:56:20,360 THERE'S NO POLITICAL WILL TO 2992 01:56:20,360 --> 01:56:21,880 SOLVE THE PROBLEM OF PRICE OF 2993 01:56:21,880 --> 01:56:23,840 GENE THERAPY, END OF STORY. 2994 01:56:23,840 --> 01:56:27,760 WE COULD TALK ABOUT THAT. 2995 01:56:27,760 --> 01:56:29,080 IT'S NOT THE NIH'S 2996 01:56:29,080 --> 01:56:31,920 RESPONSIBILITY FOR THE FDA'S 2997 01:56:31,920 --> 01:56:33,040 RESPONSIBILITY, IT'S CONGRESS' 2998 01:56:33,040 --> 01:56:33,440 RESPONSIBILITY. 2999 01:56:33,440 --> 01:56:40,680 WE CAN GET CONTROVERSIAL LATER. 3000 01:56:40,680 --> 01:56:42,640 CHALLENGE TWO, THE ROLE FOR 3001 01:56:42,640 --> 01:56:43,640 INDUSTRY. 3002 01:56:43,640 --> 01:56:46,920 I AM NOT SURE THAT PROFIT FOR 3003 01:56:46,920 --> 01:56:50,400 COMPANIES IS A GREAT MODEL FOR 3004 01:56:50,400 --> 01:56:51,400 GUARANTEEING BREAKTHROUGHS IN 3005 01:56:51,400 --> 01:56:54,480 GENE THERAPY GIVEN SO MANY 3006 01:56:54,480 --> 01:56:55,880 MONOGENICS ARE RARE DISEASES, 3007 01:56:55,880 --> 01:56:58,520 AND I LOSE SLEEP EVERY SINGLE 3008 01:56:58,520 --> 01:57:01,720 NIGHT WONDERING, BECAUSE IF WE 3009 01:57:01,720 --> 01:57:03,560 CAN'T GET THIS SUSTAINABLE, I 3010 01:57:03,560 --> 01:57:07,720 DON'T THINK ANY OF US ARE. 3011 01:57:07,720 --> 01:57:10,440 WE HAVE NEARLY FAILED MANY, MANY 3012 01:57:10,440 --> 01:57:11,560 TIMES FOR REASONS THAT HAVE 3013 01:57:11,560 --> 01:57:16,480 NOTHING TO DO WITH THE SCIENCE. 3014 01:57:16,480 --> 01:57:19,960 I DON'T KNOW IF INVESTORS AND 3015 01:57:19,960 --> 01:57:21,360 STOCKHOLDERS ARE NECESSARILY THE 3016 01:57:21,360 --> 01:57:24,120 BEST PARTNERS LONG TERM FOR OUR 3017 01:57:24,120 --> 01:57:24,560 SPACE. 3018 01:57:24,560 --> 01:57:25,080 THAT'S AN OPEN QUESTION. 3019 01:57:25,080 --> 01:57:26,800 I DON'T KNOW THE ANSWER BUT I'M 3020 01:57:26,800 --> 01:57:27,640 NOT SURE. 3021 01:57:27,640 --> 01:57:36,760 I THINK BESPOKE IS AN INCREDIBLE 3022 01:57:36,760 --> 01:57:40,160 IMPORTANT INNOVATION, I'M NOT 3023 01:57:40,160 --> 01:57:42,120 SURE IF BUSINESS SIDE OF THINGS 3024 01:57:42,120 --> 01:57:46,160 IS GOING TO BE SUSTAINABLE LONG 3025 01:57:46,160 --> 01:57:46,400 TERM. 3026 01:57:46,400 --> 01:57:48,120 CHALLENGE NUMBER THREE, I 3027 01:57:48,120 --> 01:57:50,680 MENTIONED THIS EARLIER, GENE 3028 01:57:50,680 --> 01:57:52,520 THERAPY REQUIRES A RADICAL 3029 01:57:52,520 --> 01:57:54,160 DIAGNOSTIC PARADIGM SHIFT IN 3030 01:57:54,160 --> 01:57:56,680 WHICH DOCTORS TREAT CHILDREN WHO 3031 01:57:56,680 --> 01:57:58,960 LOOK PERFECTLY HEALTHY. 3032 01:57:58,960 --> 01:58:00,640 IMAGINE WE HAVE NOW TREATED 3033 01:58:00,640 --> 01:58:02,040 THREE PRESYMPTOMATIC CHILDREN IN 3034 01:58:02,040 --> 01:58:04,000 THE LAST YEAR WITH COMMERCIALLY 3035 01:58:04,000 --> 01:58:08,960 APPROVED DRUG IN ENGLAND AND 3036 01:58:08,960 --> 01:58:09,280 GERMANY. 3037 01:58:09,280 --> 01:58:11,560 WE'VE ASKED THE BRITISH PEOPLE 3038 01:58:11,560 --> 01:58:13,240 AND GERMAN PEOPLE TO SPEND, I 3039 01:58:13,240 --> 01:58:15,000 DON'T KNOW HOW MUCH, MILLIONS OF 3040 01:58:15,000 --> 01:58:17,960 EUROS ON A BABY THAT LOOKS 3041 01:58:17,960 --> 01:58:19,400 PERFECTLY FINE. 3042 01:58:19,400 --> 01:58:22,360 THAT IS A HUGE LIFT. 3043 01:58:22,360 --> 01:58:24,000 HOW DO WE KNOW THIS BABY IS 3044 01:58:24,000 --> 01:58:27,400 GOING TO GET SICK ONE DAY? 3045 01:58:27,400 --> 01:58:29,720 AND SO THOSE CHALLENGES ARE 3046 01:58:29,720 --> 01:58:30,840 REALLY SERIOUS. 3047 01:58:30,840 --> 01:58:34,040 AND GETTING PRACTITIONERS IN THE 3048 01:58:34,040 --> 01:58:36,040 FIELD TO ABANDON A STYLE OF 3049 01:58:36,040 --> 01:58:37,160 MEDICINE THEY HAVE PRACTICED FOR 3050 01:58:37,160 --> 01:58:40,000 THOUSANDS OF YEARS TO IDENTIFY A 3051 01:58:40,000 --> 01:58:40,960 CHILD FROM A SYMPTOM 3052 01:58:40,960 --> 01:58:41,760 PRESENTATION CLINICALLY IS GOING 3053 01:58:41,760 --> 01:58:44,080 TO BE A HUGE CHALLENGE WHICH I 3054 01:58:44,080 --> 01:58:46,280 THINK THE NIH AS MUCH AS I 3055 01:58:46,280 --> 01:58:49,000 RESPECT P.J., I THINK THE NIH 3056 01:58:49,000 --> 01:58:50,640 SHOULD BE TRAINING AND TALKING 3057 01:58:50,640 --> 01:59:00,720 TO THE APA AND AMA ABOUT THE 3058 01:59:00,720 --> 01:59:02,560 TOOLS OF GENOMIC MEDICINE. 3059 01:59:02,560 --> 01:59:04,200 CHALLENGE FOUR, WHAT THE HECK 3060 01:59:04,200 --> 01:59:05,760 DOES GENE THERAPY EVEN DO? 3061 01:59:05,760 --> 01:59:07,280 THAT'S NOT A SILLY QUESTION. 3062 01:59:07,280 --> 01:59:11,320 WE DON'T KNOW WHAT IT DOES, 3063 01:59:11,320 --> 01:59:11,600 HONESTLY. 3064 01:59:11,600 --> 01:59:13,760 IT'S BEEN TEN YEARS SINCE THE 3065 01:59:13,760 --> 01:59:15,040 FIRST CHILDREN WERE TREATED IN 3066 01:59:15,040 --> 01:59:19,760 MY CLIME CLINICAL TRIAL. 3067 01:59:19,760 --> 01:59:22,240 THEY ARE WALKING AROUND, MILD 3068 01:59:22,240 --> 01:59:23,160 NEUROPATHY, SOME USE BRACES, 3069 01:59:23,160 --> 01:59:24,320 SOME DO NOT. 3070 01:59:24,320 --> 01:59:27,400 SOME HAVE VERY HIGH I.Q.s, 3071 01:59:27,400 --> 01:59:29,280 SOME MORE AVERAGE OR BELOW 3072 01:59:29,280 --> 01:59:29,720 NORMAL. 3073 01:59:29,720 --> 01:59:33,360 THEY ARE BEAUTIFUL AND AMAZING. 3074 01:59:33,360 --> 01:59:36,280 HOWEVER, WHAT IF THIS THERAPY IS 3075 01:59:36,280 --> 01:59:37,600 USED ON SYMPTOMATIC CHILDREN WHO 3076 01:59:37,600 --> 01:59:40,120 GET 20 YEARS IN A WHEELCHAIR? 3077 01:59:40,120 --> 01:59:42,200 WHAT IF IT'S USED ON PEOPLE AND 3078 01:59:42,200 --> 01:59:43,840 WE'VE OWN GIVEN THEM A MILDER 3079 01:59:43,840 --> 01:59:45,040 VERSION OF THE DISEASE AND ONE 3080 01:59:45,040 --> 01:59:50,080 DAY THEY GET SICK? 3081 01:59:50,080 --> 01:59:52,280 IN ONCOLOGY WORLD FIVE YEARS 3082 01:59:52,280 --> 01:59:54,880 DISEASE FREE IS A CURE. 3083 01:59:54,880 --> 01:59:56,120 IN RARE DISEASE SPACE THAT'S NOT 3084 01:59:56,120 --> 01:59:56,520 TRUE. 3085 01:59:56,520 --> 02:00:00,840 I NEVER USE THE C WORD, WHICH IS 3086 02:00:00,840 --> 02:00:02,360 CURE. 3087 02:00:02,360 --> 02:00:03,680 I'M MAKING A JOKE. 3088 02:00:03,680 --> 02:00:06,400 I CAN'T USE THE C WORD. 3089 02:00:06,400 --> 02:00:09,240 BUT THE CANCER COMMUNITY DOES 3090 02:00:09,240 --> 02:00:11,440 FOR CAR T CELL, WHICH IS ANOTHER 3091 02:00:11,440 --> 02:00:14,520 ISSUE I THINK WE CAN DISCUSS. 3092 02:00:14,520 --> 02:00:17,400 AND I THINK I WILL LEAVE IT 3093 02:00:17,400 --> 02:00:17,760 THERE. 3094 02:00:17,760 --> 02:00:20,960 OH, ONE LAST CHALLENGE. 3095 02:00:20,960 --> 02:00:22,600 THE THING THAT HAUNTS ME MOST OF 3096 02:00:22,600 --> 02:00:24,800 ALL IS THAT OF ALL THE CHILDREN 3097 02:00:24,800 --> 02:00:26,200 I GOT TO ITALY, THERE WAS ONLY 3098 02:00:26,200 --> 02:00:28,840 ONE CHILD I COULD NOT GET TO 3099 02:00:28,840 --> 02:00:29,680 ITALY. 3100 02:00:29,680 --> 02:00:31,360 A YOUNG AFRICAN AMERICAN MOTHER 3101 02:00:31,360 --> 02:00:36,160 AND HER BABY OUT OF RURAL NORTH 3102 02:00:36,160 --> 02:00:36,440 CAROLINA. 3103 02:00:36,440 --> 02:00:38,800 THIS IS GOING TO BE A HUGE 3104 02:00:38,800 --> 02:00:40,680 MEDICAL JUSTICE ISSUE. 3105 02:00:40,680 --> 02:00:42,720 AND THIS DISEASE, MLD, HAS A 3106 02:00:42,720 --> 02:00:45,360 HIGH INCIDENCE IN THE NAVAJO 3107 02:00:45,360 --> 02:00:48,160 NATION, AND THE ALASKA TRIBAL 3108 02:00:48,160 --> 02:00:48,680 NATION. 3109 02:00:48,680 --> 02:00:50,640 I HAVE NEVER SEEN A CHILD 3110 02:00:50,640 --> 02:00:52,400 REFERRED FROM A TRIBAL HEALTH 3111 02:00:52,400 --> 02:00:55,360 CARE SYSTEM. 3112 02:00:55,360 --> 02:00:58,280 WE CAN'T EVEN GET IN THERE. 3113 02:00:58,280 --> 02:01:01,680 RIGHT NOW THE NAVAJO NATION SHUT 3114 02:01:01,680 --> 02:01:03,000 DOWN ALL GENETIC TESTING AND 3115 02:01:03,000 --> 02:01:03,240 RESEARCH. 3116 02:01:03,240 --> 02:01:05,440 I DON'T KNOW HOW WE'LL EVER GET 3117 02:01:05,440 --> 02:01:05,680 IN. 3118 02:01:05,680 --> 02:01:07,160 THESE CHALLENGES ARE WHAT KEEP 3119 02:01:07,160 --> 02:01:08,080 ME UP AT NIGHT. 3120 02:01:08,080 --> 02:01:12,880 AND NOW I PASS IT OFF TO DOUBLE 3121 02:01:12,880 --> 02:01:17,360 DOWN AND MAKE THINGS MORE 3122 02:01:17,360 --> 02:01:19,200 INTERESTING. 3123 02:01:19,200 --> 02:01:20,320 >> GOOD AFTERNOON. 3124 02:01:20,320 --> 02:01:23,400 I SHOULD START BY SAYING HOW 3125 02:01:23,400 --> 02:01:24,600 INCREDIBLY HUMBLED I AM TO BE 3126 02:01:24,600 --> 02:01:28,520 HERE AND HAVE THE OPPORTUNITY TO 3127 02:01:28,520 --> 02:01:30,000 LISTEN TO FAMILY STORIES, 3128 02:01:30,000 --> 02:01:31,640 PATIENT STORIES, AND I THINK 3129 02:01:31,640 --> 02:01:35,000 THIS IS SUCH AN INCREDIBLE FORUM 3130 02:01:35,000 --> 02:01:37,400 WHERE MAYBE I DON'T SPEAK JUST 3131 02:01:37,400 --> 02:01:38,920 ON MYSELF, WE DON'T ALWAYS GET 3132 02:01:38,920 --> 02:01:44,600 IN A ROOM AND CAN HEAR DIRECTLY 3133 02:01:44,600 --> 02:01:55,160 ABOUT THOSE EXPERIENCES, SO I'M 3134 02:01:57,200 --> 02:01:58,560 VASILIKI RAHIMZADEH, GREEK AND 3135 02:01:58,560 --> 02:02:00,040 IRANIAN, GRADE SCHOOL WAS FUN. 3136 02:02:00,040 --> 02:02:01,920 YOU HAVE A FULL RANGE OF 3137 02:02:01,920 --> 02:02:03,360 PROFESSIONS, THE JOKE, YOU CAN 3138 02:02:03,360 --> 02:02:06,520 BE A DOCTOR, LAWYER, OR 3139 02:02:06,520 --> 02:02:06,800 ENGINEER. 3140 02:02:06,800 --> 02:02:08,960 SO, AMONG THE THREE OF US, SEEMS 3141 02:02:08,960 --> 02:02:11,440 WE'RE ALL IN THE MEDICAL 3142 02:02:11,440 --> 02:02:14,400 PROFESSION, PROBABLY NOT BY 3143 02:02:14,400 --> 02:02:14,880 COINCIDENCE. 3144 02:02:14,880 --> 02:02:17,360 MY ROLE AS A FACULTY MEMBER AT 3145 02:02:17,360 --> 02:02:18,360 BAYLOR COLLEGE OF MEDICINE, IN 3146 02:02:18,360 --> 02:02:20,560 THE CENTER FOR MEDICAL ETHICS 3147 02:02:20,560 --> 02:02:24,120 AND HEALTH POLICY, A LOT OF MY 3148 02:02:24,120 --> 02:02:26,680 RESEARCH CENTERS ON ETHICAL, 3149 02:02:26,680 --> 02:02:28,640 LEGAL, SOCIAL ISSUES OF EMERGING 3150 02:02:28,640 --> 02:02:30,160 GENETIC TECHNOLOGIES WITH 3151 02:02:30,160 --> 02:02:32,000 SPECIAL FOCUS ON CHILDREN. 3152 02:02:32,000 --> 02:02:37,280 AND I SPEND MUCH OF MY EARLY 3153 02:02:37,280 --> 02:02:41,760 CAREER FOCUSED ON SOME OF THE 3154 02:02:41,760 --> 02:02:43,280 DATA GOVERNANCE AND PRIVACY 3155 02:02:43,280 --> 02:02:45,680 ISSUES AND SHARING GENOMIC AND 3156 02:02:45,680 --> 02:02:46,680 ASSOCIATE DATA WITH CHILDREN, 3157 02:02:46,680 --> 02:02:49,320 THE RARE DISEASE COMMUNITY IS 3158 02:02:49,320 --> 02:02:52,800 OFTEN A POSTER CHILD, A POSTER 3159 02:02:52,800 --> 02:02:55,320 DISEASE NETWORK IN WHICH GENOMIC 3160 02:02:55,320 --> 02:02:58,600 DATA SHARING IS NOT ONLY 3161 02:02:58,600 --> 02:02:59,520 ADVANCING SCIENTIFIC DISCOVERY 3162 02:02:59,520 --> 02:03:02,680 BUT OFTEN WHERE INFORMATION AND 3163 02:03:02,680 --> 02:03:04,000 DATA ARE ACTUALLY THE TREATMENT 3164 02:03:04,000 --> 02:03:05,560 BECAUSE OF ITS RARITY, BECAUSE 3165 02:03:05,560 --> 02:03:10,840 OF THE SMALL N AND THE NEED, 3166 02:03:10,840 --> 02:03:13,560 SCIENTIFIC NEED TO IDENTIFY 3167 02:03:13,560 --> 02:03:16,480 PATIENTS WITH SIMILAR CLINICAL 3168 02:03:16,480 --> 02:03:18,360 AND GENOTYPE -- PHENOTYPE AND 3169 02:03:18,360 --> 02:03:21,080 GENOTYPE TO LINK THEM TOGETHER 3170 02:03:21,080 --> 02:03:24,080 TO DEVELOP AND FIND AND ENHANCE 3171 02:03:24,080 --> 02:03:26,240 DATA DISCOVERY ACROSS THESE 3172 02:03:26,240 --> 02:03:28,760 DIFFERENT CASES TO EXPAND KIND 3173 02:03:28,760 --> 02:03:31,160 OF THE EVIDENCE BASE. 3174 02:03:31,160 --> 02:03:33,880 AND SO THIS COMMUNITY NOT ONLY 3175 02:03:33,880 --> 02:03:35,320 BEING A SCIENTIFIC IMPERATIVE TO 3176 02:03:35,320 --> 02:03:36,640 DO THAT, THERE ARE ETHICAL 3177 02:03:36,640 --> 02:03:38,280 CONSEQUENCES TO A LOT OF THE 3178 02:03:38,280 --> 02:03:40,560 SCIENCE WE'RE TALKING ABOUT 3179 02:03:40,560 --> 02:03:40,760 TODAY. 3180 02:03:40,760 --> 02:03:43,080 SO I THOUGHT IT WOULD BE HELPFUL 3181 02:03:43,080 --> 02:03:45,840 TO OPEN UP, WE STARTED A 3182 02:03:45,840 --> 02:03:46,680 CONVERSATION ABOUT NEWBORN 3183 02:03:46,680 --> 02:03:49,880 SCREENING, THIS IS AN AREA IN 3184 02:03:49,880 --> 02:03:52,080 WHICH THERE'S BEEN DEBATE AND 3185 02:03:52,080 --> 02:03:54,280 CONTROVERSY, MOSTLY ON ETHICS, 3186 02:03:54,280 --> 02:03:56,120 TO INTEGRATING WHOLE GENOME AND 3187 02:03:56,120 --> 02:03:57,240 EXOME SEQUENCING AT THE NEWBORN 3188 02:03:57,240 --> 02:03:59,120 SCREENING LEVEL. 3189 02:03:59,120 --> 02:04:00,520 I THOUGHT I WOULD ANIMATE SOME 3190 02:04:00,520 --> 02:04:04,120 OF THAT DISCUSSION AS PROMISED. 3191 02:04:04,120 --> 02:04:06,960 AND SO THE FIRST -- I'LL TRY TO 3192 02:04:06,960 --> 02:04:08,760 MAYBE LANDSCAPE SOME OF THE 3193 02:04:08,760 --> 02:04:13,000 ETHICAL DEBATE ON BOTH SIDES. 3194 02:04:13,000 --> 02:04:16,400 THE FIRST IS THAT, YOU KNOW, 3195 02:04:16,400 --> 02:04:18,040 PUBLIC HEALTH SCREENING PROGRAMS 3196 02:04:18,040 --> 02:04:20,600 LIKE NEWBORN SCREENING IS MEANT 3197 02:04:20,600 --> 02:04:23,560 TO -- IT HAS THIS POPULATION 3198 02:04:23,560 --> 02:04:24,640 HEALTH FOCUS. 3199 02:04:24,640 --> 02:04:27,080 AND IT IS SOMEWHAT ANTAGONISTIC 3200 02:04:27,080 --> 02:04:29,680 TO THE WHOLE GENOME AND EXOME 3201 02:04:29,680 --> 02:04:30,680 SEQUENCING MANTRA, WHICH IS VERY 3202 02:04:30,680 --> 02:04:32,920 MUCH PRECISION MEDICINE, VERY 3203 02:04:32,920 --> 02:04:34,520 MUCH PERSONALIZED. 3204 02:04:34,520 --> 02:04:37,520 AND WHEN WE LOOK ACROSS PLACES, 3205 02:04:37,520 --> 02:04:39,920 EVEN WITHIN THIS COUNTRY, 3206 02:04:39,920 --> 02:04:40,920 NEWBORN SCREENING IS 3207 02:04:40,920 --> 02:04:41,880 DIFFERENTIALLY APPLIED. 3208 02:04:41,880 --> 02:04:44,720 IT'S DIFFERENTIALLY COVERED. 3209 02:04:44,720 --> 02:04:46,440 IT'S DIFFERENTIALLY EFFECTIVE. 3210 02:04:46,440 --> 02:04:50,400 AND THIS IS VERY MUCH AT THE 3211 02:04:50,400 --> 02:04:51,760 GOVERNMENT AND STATE RESOURCING 3212 02:04:51,760 --> 02:04:55,440 LEVEL THAT'S HAVING SUCH 3213 02:04:55,440 --> 02:04:56,440 DIFFERENT OUTCOMES, KIND OF AT 3214 02:04:56,440 --> 02:04:58,000 THE GROUND LEVEL. 3215 02:04:58,000 --> 02:05:01,280 WHEN WE SCALE TO INTERNATIONAL 3216 02:05:01,280 --> 02:05:02,680 CONTEXT, IT'S RATHER ABYSMAL. 3217 02:05:02,680 --> 02:05:04,120 AMONG THE PUBLIC HEALTH 3218 02:05:04,120 --> 02:05:07,400 COMMUNITY AND PUBLIC HEALTH 3219 02:05:07,400 --> 02:05:09,720 GENOMICS PROFESSIONALS ARE 3220 02:05:09,720 --> 02:05:11,240 PASSIONATE ABOUT ENSURING BASIC 3221 02:05:11,240 --> 02:05:12,560 NEWBORN SCREENING THAT'S 3222 02:05:12,560 --> 02:05:14,400 AVAILABLE ACROSS ALL POPULATIONS 3223 02:05:14,400 --> 02:05:17,240 FOR EVERY BABY, EVERYWHERE, AND 3224 02:05:17,240 --> 02:05:18,640 THIS HAS NOT HISTORICALLY BEEN 3225 02:05:18,640 --> 02:05:21,600 ACHIEVED TO DATE. 3226 02:05:21,600 --> 02:05:25,520 SO ADDING IN REALLY EXPENSIVE, 3227 02:05:25,520 --> 02:05:28,720 VERY HIGH RESOURCE, BOTH HUMAN 3228 02:05:28,720 --> 02:05:29,280 AND MATERIAL RESOURCE 3229 02:05:29,280 --> 02:05:32,240 INTENSIVENESS TO THE NEWBORN 3230 02:05:32,240 --> 02:05:34,880 SCREENING PROCESS IS GOING TO 3231 02:05:34,880 --> 02:05:37,520 EXACERBATE THESE ISSUES WE KNOW 3232 02:05:37,520 --> 02:05:39,800 CONTINUE TODAY. 3233 02:05:39,800 --> 02:05:42,200 SO YOU HEAR AND ARE FACED WITH 3234 02:05:42,200 --> 02:05:44,800 PATIENTS AND FAMILIES FOR WHOM 3235 02:05:44,800 --> 02:05:46,720 WHOLE GENOME AND EXOME 3236 02:05:46,720 --> 02:05:50,080 SEQUENCING MADE AN INCREDIBLE 3237 02:05:50,080 --> 02:05:52,960 IMPACT ON DIAGNOSTIC YIELD, ON 3238 02:05:52,960 --> 02:05:55,120 ENDING THE DIAGNOSTIC ODYSSEY 3239 02:05:55,120 --> 02:05:57,640 FOR MANY PATIENTS AND FAMILIES 3240 02:05:57,640 --> 02:06:00,240 AND VERY DIFFICULT TO ARGUE 3241 02:06:00,240 --> 02:06:01,920 THAT, YOU KNOW, THAT ACCESS 3242 02:06:01,920 --> 02:06:03,880 WASN'T A DIRECT CONSEQUENCE, 3243 02:06:03,880 --> 02:06:06,720 THAT DIAGNOSTIC -- THAT 3244 02:06:06,720 --> 02:06:08,000 DIAGNOSIS ITSELF, ACCURATE 3245 02:06:08,000 --> 02:06:09,680 DIAGNOSIS, WASN'T A DIRECT 3246 02:06:09,680 --> 02:06:12,000 CONSEQUENCE OF SEQUENCING AND I 3247 02:06:12,000 --> 02:06:14,320 THINK THAT'S SUCH A PASSIONATE 3248 02:06:14,320 --> 02:06:15,840 APPEAL FOR PATIENTS AND FAMILIES 3249 02:06:15,840 --> 02:06:23,040 AND WHAT'S REALLY DRIVING THE 3250 02:06:23,040 --> 02:06:25,880 CONVERSATION FOR AT LEAST 3251 02:06:25,880 --> 02:06:26,880 STUDYING IMPLEMENTATION AT 3252 02:06:26,880 --> 02:06:27,880 NEWBORN SCREENING LEVEL. 3253 02:06:27,880 --> 02:06:29,000 WE KNOW FOR OTHER CONDITIONS 3254 02:06:29,000 --> 02:06:31,040 THERE ARE MANY FOR WHICH WE HAVE 3255 02:06:31,040 --> 02:06:35,280 A LOT OF DATA TO SUGGEST THE 3256 02:06:35,280 --> 02:06:36,960 CLINICAL ASSOCIATION AND GENETIC 3257 02:06:36,960 --> 02:06:40,160 AND GENOMIC ETIOLOGY OF DISEASE. 3258 02:06:40,160 --> 02:06:42,600 THERE ARE LOTS WE KNOW VERY 3259 02:06:42,600 --> 02:06:44,440 LITTLE ABOUT, CONTINUOUSLY 3260 02:06:44,440 --> 02:06:44,800 CHANGING. 3261 02:06:44,800 --> 02:06:47,280 THERE'S PROBABLY MORE VARIANTS 3262 02:06:47,280 --> 02:06:49,160 OF UNKNOWN SIGNIFICANCE THAN 3263 02:06:49,160 --> 02:06:50,800 THOSE VARIANTS OF KNOWN 3264 02:06:50,800 --> 02:06:51,880 SIGNIFICANCE. 3265 02:06:51,880 --> 02:06:53,240 AND THIS HAS IMPORTANT 3266 02:06:53,240 --> 02:06:53,960 IMPLICATIONS FOR CLINICAL 3267 02:06:53,960 --> 02:06:55,080 DECISION MAKING, WHEN WE'RE 3268 02:06:55,080 --> 02:06:57,680 FACED WITH A FAMILY WHO HAS TO 3269 02:06:57,680 --> 02:07:00,880 DECIDE TREATMENT A, TREATMENT B, 3270 02:07:00,880 --> 02:07:03,960 TREATMENT C, AND THIS VARIANT 3271 02:07:03,960 --> 02:07:05,440 WE'VE DISCOVERED IS EITHER 3272 02:07:05,440 --> 02:07:07,640 BENIGN TODAY OR UNKNOWN TODAY, 3273 02:07:07,640 --> 02:07:09,920 AND THAT HAS CHANGES. 3274 02:07:09,920 --> 02:07:12,640 THERE'S BEEN A LOT OF DISCUSSION 3275 02:07:12,640 --> 02:07:13,920 IN THE CLINICAL AND 3276 02:07:13,920 --> 02:07:16,440 BIOINFORMATICS COMMUNITY ABOUT 3277 02:07:16,440 --> 02:07:19,080 HOW WE ACTUALLY MERGE RESEARCH 3278 02:07:19,080 --> 02:07:22,040 DATA AND EMERGING, YOU KNOW, 3279 02:07:22,040 --> 02:07:23,080 SCIENTIFIC DATA INTO THE 3280 02:07:23,080 --> 02:07:25,120 CLINICAL REALM AND SOME OF OUR 3281 02:07:25,120 --> 02:07:25,880 EXISTING DATA INFRASTRUCTURES 3282 02:07:25,880 --> 02:07:31,000 DON'T ALLOW FOR THAT. 3283 02:07:31,000 --> 02:07:34,760 SO THERE IS A WHOLE ECOSYSTEM, 3284 02:07:34,760 --> 02:07:36,000 UPDATING THAT DOESN'T CURRENTLY 3285 02:07:36,000 --> 02:07:38,040 HAPPEN, THAT HAS VERY IMPORTANT 3286 02:07:38,040 --> 02:07:39,240 IMPLICATIONS FOR DECISIONS AT 3287 02:07:39,240 --> 02:07:40,240 THE BEDSIDE. 3288 02:07:40,240 --> 02:07:43,720 SO THESE ARE JUST SOME ARGUMENTS 3289 02:07:43,720 --> 02:07:44,920 FOR AND AGAINST, AND AT THE 3290 02:07:44,920 --> 02:07:49,080 MOMENT ONE OF THE MOST IMPORTANT 3291 02:07:49,080 --> 02:07:51,160 EXPERIMENTS, I'LL CALL IT, PILOT 3292 02:07:51,160 --> 02:07:54,960 TESTING THAT'S REALLY HAPPENING, 3293 02:07:54,960 --> 02:07:57,080 IS THROUGH THE NATIONAL HEALTH 3294 02:07:57,080 --> 02:08:00,240 SERVICE IN THE UNITED KINGDOM 3295 02:08:00,240 --> 02:08:01,760 PILOTING WHOLE GENOME SEQUENCING 3296 02:08:01,760 --> 02:08:03,840 WITHIN THEIR POPULATION AND IT 3297 02:08:03,840 --> 02:08:07,600 HAS BUILT IN AN EVALUATION 3298 02:08:07,600 --> 02:08:08,760 STUDY, A LONG-TERM OUTCOME STUDY 3299 02:08:08,760 --> 02:08:11,960 TO SEE WHEN WE DO THIS AT A 3300 02:08:11,960 --> 02:08:13,640 POPULATION LEVEL AND GETTING THE 3301 02:08:13,640 --> 02:08:15,240 NUMBERS WE NEED AND FOR OTHER 3302 02:08:15,240 --> 02:08:16,800 COUNTRIES TO REALLY FOLLOW SUIT 3303 02:08:16,800 --> 02:08:19,840 AND GET DATA ON HOW DOES THIS -- 3304 02:08:19,840 --> 02:08:22,480 HOW IS THIS OPERATIONALIZED IN A 3305 02:08:22,480 --> 02:08:24,760 POPULATION LEVEL, WHAT IS THE, 3306 02:08:24,760 --> 02:08:26,080 YOU KNOW, AT THE POINT OF CARE 3307 02:08:26,080 --> 02:08:29,120 WHAT ARE THE IMPACTS TO CLINICAL 3308 02:08:29,120 --> 02:08:31,760 DECISIONMAKING, AND THEN AT 3309 02:08:31,760 --> 02:08:34,200 LEAST WITH, YOU KNOW, A LOT OF 3310 02:08:34,200 --> 02:08:35,960 IMPORTANCE AND TIMELINESS AND 3311 02:08:35,960 --> 02:08:38,920 SENSITIVITY, IN THIS COUNTRY, 3312 02:08:38,920 --> 02:08:40,400 THE IMPACTS ON REPRODUCTIVE 3313 02:08:40,400 --> 02:08:41,760 DECISION MAKING. 3314 02:08:41,760 --> 02:08:45,360 WE'RE LIVING IN A POST-ROWE ERA, 3315 02:08:45,360 --> 02:08:47,520 OVERNIGHT A LOT OF THESE 3316 02:08:47,520 --> 02:08:48,520 REPRODUCTIVE DECISIONS THAT WERE 3317 02:08:48,520 --> 02:08:50,640 AVAILABLE JUST A YEAR AGO ARE 3318 02:08:50,640 --> 02:08:51,640 NOT TODAY. 3319 02:08:51,640 --> 02:08:55,040 AND I THINK THAT ADDS ANOTHER 3320 02:08:55,040 --> 02:08:57,080 LAYER OF ETHICAL COMPLEXITY TO 3321 02:08:57,080 --> 02:09:00,040 SOME OF THESE NEWBORN SCREENING 3322 02:09:00,040 --> 02:09:01,680 DECISIONS. 3323 02:09:01,680 --> 02:09:04,120 I COULD TALK AT LENGTH AND I'LL 3324 02:09:04,120 --> 02:09:05,400 END A LITTLE BIT WITH SOME 3325 02:09:05,400 --> 02:09:09,360 ISSUES AND DATA THAT I'M FOCUSED 3326 02:09:09,360 --> 02:09:14,480 ON, SO THERE ARE A FIELD OF 3327 02:09:14,480 --> 02:09:15,240 ETHICS AND PEDIATRIC 3328 02:09:15,240 --> 02:09:16,160 BIOETHICISTS CONCERNED ABOUT 3329 02:09:16,160 --> 02:09:18,440 FUTURE PRIVACY OF CHILDREN. 3330 02:09:18,440 --> 02:09:19,840 AS WE'RE ADVOCATING FOR OPEN 3331 02:09:19,840 --> 02:09:25,680 DATA TRANSPARENCY IN THE DATA, 3332 02:09:25,680 --> 02:09:27,200 MERGING DATASETS TOGETHER, WE'RE 3333 02:09:27,200 --> 02:09:29,600 INTERESTED WHAT EFFECT THAT WILL 3334 02:09:29,600 --> 02:09:31,160 HAVE BOTH HARMFUL AND BENEFICIAL 3335 02:09:31,160 --> 02:09:34,240 TO CHILDREN AS THEY GET OLDER, 3336 02:09:34,240 --> 02:09:36,400 WILL THEY MAKE THE SAME DATA 3337 02:09:36,400 --> 02:09:37,760 DECISIONS AND DATA SHARING 3338 02:09:37,760 --> 02:09:40,160 DECISIONS AS THEIR PARENTS? 3339 02:09:40,160 --> 02:09:42,320 AND WILL THEY HAVE THIS CONCEPT 3340 02:09:42,320 --> 02:09:44,160 CALLED A RIGHT TO OPEN FUTURE, 3341 02:09:44,160 --> 02:09:46,280 RIGHT TO A PRIVATE FUTURE, IN 3342 02:09:46,280 --> 02:09:48,000 MAKING SOME OF THOSE DECISIONS 3343 02:09:48,000 --> 02:09:51,000 ONCE THEY REACH THE AGE OF 3344 02:09:51,000 --> 02:09:51,760 MAJORITY. 3345 02:09:51,760 --> 02:09:54,680 THOSE ARE SOME ETHICAL AND 3346 02:09:54,680 --> 02:09:56,120 PHILOSOPHICAL THINGS I'VE BEEN 3347 02:09:56,120 --> 02:10:00,640 FOCUSED ON IN MY WORK. 3348 02:10:00,640 --> 02:10:01,400 >> THANK YOU. 3349 02:10:01,400 --> 02:10:03,680 I HAVE A COUPLE QUESTIONS AND 3350 02:10:03,680 --> 02:10:07,960 WE'LL BE TAKING QUESTIONS FROM 3351 02:10:07,960 --> 02:10:09,920 THE AUDIENCE. 3352 02:10:09,920 --> 02:10:16,840 STARTING WITH REGHAN, HOW DO YOU 3353 02:10:16,840 --> 02:10:18,080 ENSURE TRULY INFORMED CONSENT, 3354 02:10:18,080 --> 02:10:21,680 RISKS ASSOCIATED WITH GENE 3355 02:10:21,680 --> 02:10:25,080 THERAPY, CLINICAL TRIALS? 3356 02:10:25,080 --> 02:10:27,840 >> FOR ME, THIS IS A VERY 3357 02:10:27,840 --> 02:10:28,200 IMPORTANT TOPIC. 3358 02:10:28,200 --> 02:10:30,080 AS A CLINICIAN, A PHYSICIAN, 3359 02:10:30,080 --> 02:10:32,520 WE'RE THE ONES WHO ARE OBTAINING 3360 02:10:32,520 --> 02:10:33,480 CONSENT FROM FAMILIES AND 3361 02:10:33,480 --> 02:10:35,480 PARENTS PRIOR TO GENE THERAPY 3362 02:10:35,480 --> 02:10:37,440 DELIVERY. 3363 02:10:37,440 --> 02:10:38,760 I THINK IT'S ESSENTIAL, TO BE 3364 02:10:38,760 --> 02:10:40,400 TRULY INFORMED THE PHYSICIAN HAS 3365 02:10:40,400 --> 02:10:43,440 TO BE REALLY AWARE IN REAL TIME 3366 02:10:43,440 --> 02:10:45,120 OF ALL THE ADVERSE EVENTS WITH 3367 02:10:45,120 --> 02:10:45,960 THE GENE THERAPY PRODUCT AROUND 3368 02:10:45,960 --> 02:10:46,760 THE WORLD. 3369 02:10:46,760 --> 02:10:49,000 IF THE PHYSICIAN IS NOT AWARE OF 3370 02:10:49,000 --> 02:10:55,000 ALL THE EVENTS AT THE TIME OF 3371 02:10:55,000 --> 02:10:56,920 CONSENT, IT'S NOT TRULY INFORMED 3372 02:10:56,920 --> 02:10:57,160 CONSENT. 3373 02:10:57,160 --> 02:10:59,680 GOING BACK TO TRANSPARENCY WITH 3374 02:10:59,680 --> 02:11:00,440 SPONSORS, INVESTIGATORS, ALL 3375 02:11:00,440 --> 02:11:01,360 INVESTIGATORS NEED A FORUM TO 3376 02:11:01,360 --> 02:11:03,800 SHARE IN REAL TIME WHAT THEY ARE 3377 02:11:03,800 --> 02:11:04,960 SEEING BECAUSE THEY ARE USUALLY 3378 02:11:04,960 --> 02:11:07,280 THE EXPERTS IN THAT RARE DISEASE 3379 02:11:07,280 --> 02:11:10,320 AND THE SPONSOR MAY NOT BE AS 3380 02:11:10,320 --> 02:11:12,120 EXPERT OR ALARMED, BUT THE 3381 02:11:12,120 --> 02:11:21,080 FINDING WOULD BE ALARMING TO THE 3382 02:11:21,080 --> 02:11:21,360 PHYSICIAN. 3383 02:11:21,360 --> 02:11:22,400 IT INCLUDES HONEST DISCUSSION 3384 02:11:22,400 --> 02:11:23,800 ABOUT FAMILIES ABOUT WHAT IS 3385 02:11:23,800 --> 02:11:26,320 KNOWN IN THE STUDY SO FAR AS 3386 02:11:26,320 --> 02:11:28,480 WELL AS UNKNOWNS, INCLUDING 3387 02:11:28,480 --> 02:11:28,680 DEATH. 3388 02:11:28,680 --> 02:11:36,040 IT IS ONLY HUMAN NATURE, IT'S O 3389 02:11:36,040 --> 02:11:37,320 BE EXPECTED SUBCONSCIOUSLY 3390 02:11:37,320 --> 02:11:38,440 EXPECT A POSITIVE OUTCOMES, AND 3391 02:11:38,440 --> 02:11:40,080 THEY ARE THE ONES THAT ARE 3392 02:11:40,080 --> 02:11:42,720 REJECT THE AS BEING THE OUTCOME 3393 02:11:42,720 --> 02:11:50,120 EVERYONE IS SEEING BUT NOT EVERY 3394 02:11:50,120 --> 02:11:51,760 OUTCOME IS POSITIVE THE FAMILY 3395 02:11:51,760 --> 02:11:53,560 HAS TO UNDERSTAND ONE POSSIBLE 3396 02:11:53,560 --> 02:11:54,400 OUTCOME COULD BE DEATH. 3397 02:11:54,400 --> 02:11:56,400 AND THAT WOULD BE ABSOLUTELY 3398 02:11:56,400 --> 02:11:57,600 ESSENTIAL THAT FAMILY 3399 02:11:57,600 --> 02:11:58,920 UNDERSTANDS THAT AND STATES THAT 3400 02:11:58,920 --> 02:12:03,160 BEFORE THEY SIGN THE CONSENT 3401 02:12:03,160 --> 02:12:03,360 FORM. 3402 02:12:03,360 --> 02:12:07,360 FOR ME, TRULY INFORMED CONSENT 3403 02:12:07,360 --> 02:12:08,440 IS REALLY ESSENTIAL PROCESS, 3404 02:12:08,440 --> 02:12:09,760 WHAT'S KNOWN TO DATE, 3405 02:12:09,760 --> 02:12:10,440 RECOGNIZING SOMETIMES GENE 3406 02:12:10,440 --> 02:12:11,880 THERAPY CAN RESULT IN DEATH. 3407 02:12:11,880 --> 02:12:14,360 WE'VE SEEN THAT FIRST HAND, IT'S 3408 02:12:14,360 --> 02:12:17,520 VERY IMPORTANT, THIS DELICATE 3409 02:12:17,520 --> 02:12:18,160 DISCUSSION, ALSO ONGOING 3410 02:12:18,160 --> 02:12:20,040 DISCUSSION TO BE -- TO OCCUR 3411 02:12:20,040 --> 02:12:21,360 EVEN THE NIGHT BEFORE AND THE 3412 02:12:21,360 --> 02:12:23,000 DAY OF THE GENE THERAPY, DO YOU 3413 02:12:23,000 --> 02:12:25,520 STILL WANT TO PROCEED, DO YOU 3414 02:12:25,520 --> 02:12:26,200 UNDERSTAND POTENTIALLY THIS MAY 3415 02:12:26,200 --> 02:12:27,600 RESULT IN THE DEATH OF YOUR 3416 02:12:27,600 --> 02:12:31,840 CHILD, ARE YOU OKAY WITH THAT 3417 02:12:31,840 --> 02:12:34,800 RISK? 3418 02:12:34,800 --> 02:12:37,200 >> DOES ANYBODY ELSE HAVE 3419 02:12:37,200 --> 02:12:38,160 ANYTHING TO ADD? 3420 02:12:38,160 --> 02:12:41,640 >> I THINK, YOU KNOW, THE 3421 02:12:41,640 --> 02:12:44,040 TOLERANCE FOR RISK IS SO VARIED 3422 02:12:44,040 --> 02:12:46,880 AND HAVE YOU FAMILIES WHO AS 3423 02:12:46,880 --> 02:12:48,720 TERRY TALKED ABOUT SELL THEIR 3424 02:12:48,720 --> 02:12:51,360 HOUSES OR REFINANCE THEIR HOMES, 3425 02:12:51,360 --> 02:12:55,080 SO THERE'S A GREAT DEAL AT STAKE 3426 02:12:55,080 --> 02:12:57,960 IN TERMS OF -- I'VE SEEN 3427 02:12:57,960 --> 02:12:58,960 FAMILIES SELL THEIR BELONGINGS 3428 02:12:58,960 --> 02:13:01,440 AND GET ON A PLANE, SIGHT 3429 02:13:01,440 --> 02:13:04,400 UNSEEN, JUST SHOW UP AT A 3430 02:13:04,400 --> 02:13:05,280 CLINICIAN'S DOOR. 3431 02:13:05,280 --> 02:13:07,240 WE'RE NOT TERRIBLY GOOD AT 3432 02:13:07,240 --> 02:13:09,800 MAKING RATIONAL DECISIONS BUT I 3433 02:13:09,800 --> 02:13:13,600 DO THINK IT'S QUITE IMPORTANT. 3434 02:13:13,600 --> 02:13:16,440 AT THE SAME TIME, I WAS REALLY 3435 02:13:16,440 --> 02:13:18,200 IMPRESSED BY A RECENT PIECE IN 3436 02:13:18,200 --> 02:13:20,160 THE NEW YORKER ABOUT PROVIDING 3437 02:13:20,160 --> 02:13:26,280 THERAPY TO PEOPLE WHO ARE DYING. 3438 02:13:26,280 --> 02:13:27,480 MY PERSPECTIVE AS PATIENT 3439 02:13:27,480 --> 02:13:30,880 ADVOCATE IN NEUROLOGY SPACE IS 3440 02:13:30,880 --> 02:13:34,920 THAT SWING FOR THE FENCES BUT I 3441 02:13:34,920 --> 02:13:35,800 UNDERSTAND LEGITIMATE CONCERNS, 3442 02:13:35,800 --> 02:13:37,480 BUT I DEAL WITH FAMILIES MAKING 3443 02:13:37,480 --> 02:13:40,000 THIS CHOICE I TELL THEM TWO 3444 02:13:40,000 --> 02:13:40,200 THINGS. 3445 02:13:40,200 --> 02:13:41,200 ONE, IT'S A CLINICAL TRIAL. 3446 02:13:41,200 --> 02:13:45,040 YOU DON'T HAVE TO DO THIS. 3447 02:13:45,040 --> 02:13:47,280 YOU'RE NOT ABANDONING YOUR CHILD 3448 02:13:47,280 --> 02:13:50,240 IF YOU DO NOT DO WHAT TERRY DID 3449 02:13:50,240 --> 02:13:51,440 AND START A COMPANY AND YOU 3450 02:13:51,440 --> 02:13:52,760 DON'T HAVE TO DO THIS. 3451 02:13:52,760 --> 02:13:54,680 THE SECOND THING I SAY TO THEM 3452 02:13:54,680 --> 02:13:56,400 IS THAT YOU NEED TO BE ABLE TO 3453 02:13:56,400 --> 02:14:01,320 LIVE WITH WHATEVER DECISION YOU 3454 02:14:01,320 --> 02:14:01,520 MAKE. 3455 02:14:01,520 --> 02:14:02,880 WHATEVER YOU'RE CHOOSING NOW FOR 3456 02:14:02,880 --> 02:14:04,320 YOUR FAMILY, YOUR CHILD, YOU 3457 02:14:04,320 --> 02:14:06,280 NEED TO BE ABLE TO LOOK BACK AND 3458 02:14:06,280 --> 02:14:08,440 SAY I'M AT PEACE WITH THAT 3459 02:14:08,440 --> 02:14:08,720 DECISION. 3460 02:14:08,720 --> 02:14:11,160 THAT'S MY TWO CENTS FROM THE 3461 02:14:11,160 --> 02:14:11,600 PATIENT PERSPECTIVE. 3462 02:14:11,600 --> 02:14:15,240 >> I'LL BUILD A LITTLE BIT. 3463 02:14:15,240 --> 02:14:18,320 THIS PHENOMENON AND OBSERVATION 3464 02:14:18,320 --> 02:14:21,240 WE'RE MAKING ABOUT OFTEN SEEING 3465 02:14:21,240 --> 02:14:22,560 CLINICAL CARE AND RESEARCH BE 3466 02:14:22,560 --> 02:14:24,440 ONE AND THE SAME, PARTICULARLY 3467 02:14:24,440 --> 02:14:26,520 IN POPULATIONS WHERE THERE 3468 02:14:26,520 --> 02:14:28,480 AREN'T THERAPEUTIC OR 3469 02:14:28,480 --> 02:14:30,880 FDA-APPROVED THERAPIES, AND 3470 02:14:30,880 --> 02:14:34,800 CURATIVE OPTIONS, THIS IDEA OF 3471 02:14:34,800 --> 02:14:36,040 THERAPEUTIC MISCONCEPTION. 3472 02:14:36,040 --> 02:14:38,200 SO WHEN ESPECIALLY PATIENTS AND 3473 02:14:38,200 --> 02:14:39,280 FAMILIES ARE ENROLLING IN 3474 02:14:39,280 --> 02:14:42,040 CLINICAL TRIALS, THIS IS NOT 3475 02:14:42,040 --> 02:14:44,440 UNIQUE TO GENE THERAPY, YOU HAVE 3476 02:14:44,440 --> 02:14:46,560 FAMILIES THAT ARE REALLY AT THE 3477 02:14:46,560 --> 02:14:49,720 END OF THEIR ROPE, AT THE END 3478 02:14:49,720 --> 02:14:52,040 OF -- HAVE EXHAUSTED OTHER 3479 02:14:52,040 --> 02:14:52,440 OPTIONS. 3480 02:14:52,440 --> 02:14:55,520 THERE IS THIS VERY PASSIONATE 3481 02:14:55,520 --> 02:14:57,120 APPEAL TO WHATEVER IS BEING 3482 02:14:57,120 --> 02:14:58,680 PROVIDED IN THE CLINICAL TRIAL 3483 02:14:58,680 --> 02:15:01,840 AND ACCESS TO THE CLINICAL TRIAL 3484 02:15:01,840 --> 02:15:03,800 AS A MEANS OF A CURE OR AT LEAST 3485 02:15:03,800 --> 02:15:06,440 WILL GET YOU ON THE PATH TO 3486 02:15:06,440 --> 02:15:08,520 CLINICAL CARE. 3487 02:15:08,520 --> 02:15:10,920 SO WE -- THERE'S A LOT OF 3488 02:15:10,920 --> 02:15:13,120 ETHICAL DEBATE ABOUT THIS 3489 02:15:13,120 --> 02:15:14,240 CONCEPT OF THERAPEUTIC 3490 02:15:14,240 --> 02:15:16,080 MISCONCEPTION THAT HAS DIRECT 3491 02:15:16,080 --> 02:15:19,920 IMPACT ON THE INTEGRITY OF THE 3492 02:15:19,920 --> 02:15:22,320 INFORMED CONSENT PROCESS. 3493 02:15:22,320 --> 02:15:23,280 AGAIN EMPHASIZING DIFFERENCES 3494 02:15:23,280 --> 02:15:26,040 BETWEEN CLINICAL CARE AND 3495 02:15:26,040 --> 02:15:27,240 RESEARCH. 3496 02:15:27,240 --> 02:15:30,280 RESEARCH IS MEANT TO IDENTIFY 3497 02:15:30,280 --> 02:15:34,240 AND GENERATE NEW KNOWLEDGE ABOUT 3498 02:15:34,240 --> 02:15:36,080 CERTAIN BIOLOGICS AND 3499 02:15:36,080 --> 02:15:36,520 INTERVENTIONS. 3500 02:15:36,520 --> 02:15:39,040 AND YOU CAN IMAGINE THE TIMING 3501 02:15:39,040 --> 02:15:40,120 OF THAT CONVERSATION HAVING ALL 3502 02:15:40,120 --> 02:15:45,160 OF THE -- MAKING ALL OF THE 3503 02:15:45,160 --> 02:15:45,600 DIFFERENCE. 3504 02:15:45,600 --> 02:15:47,920 SO, ADDRESSING ISSUES OF 3505 02:15:47,920 --> 02:15:49,760 THERAPEUTIC MISCONCEPTION, 3506 02:15:49,760 --> 02:15:51,720 TIMING OF DISCUSSION, ANDS 3507 02:15:51,720 --> 02:15:53,160 SOMETIMES A LESSER KNOWN ASPECT 3508 02:15:53,160 --> 02:15:54,800 OF THE INFORMED CONSENT PROCESS 3509 02:15:54,800 --> 02:16:02,040 IS THAT IT'S NOT A ONE-TIME ONE, 3510 02:16:02,040 --> 02:16:03,680 YOU KNOW, DECISION TIME POINT. 3511 02:16:03,680 --> 02:16:08,400 IT SHOULD BE AN ONGOING PROCESS 3512 02:16:08,400 --> 02:16:10,040 AT DIFFERENT PARTS OF THE 3513 02:16:10,040 --> 02:16:11,440 PROCESS, SO INITIAL DISCUSSIONS 3514 02:16:11,440 --> 02:16:15,280 ABOUT WHAT ARE POSSIBLE TRIALS, 3515 02:16:15,280 --> 02:16:19,320 A FAMILY MIGHT BE ELIGIBLE FOR, 3516 02:16:19,320 --> 02:16:20,640 ACTUAL CONSULTATION PROCESS THE 3517 02:16:20,640 --> 02:16:24,000 DAY BEFORE IT HAPPENS, THIS IS A 3518 02:16:24,000 --> 02:16:25,880 DEMONSTRATION OF ONGOING AND 3519 02:16:25,880 --> 02:16:33,040 DYNAMIC CONSENT, WHICH WE HAVE 3520 02:16:33,040 --> 02:16:36,320 FOUND IN SOME STUDY ALLOWS FOR 3521 02:16:36,320 --> 02:16:38,840 RIGOROUS AND INFORMED CONSENT 3522 02:16:38,840 --> 02:16:39,080 PROCESS. 3523 02:16:39,080 --> 02:16:45,200 ONE OF THE OTHER MAJOR ISSUES 3524 02:16:45,200 --> 02:16:48,320 THAT WE'RE FACING IS THE 3525 02:16:48,320 --> 02:16:49,360 DIVERSIFICATION WITHIN DATASETS 3526 02:16:49,360 --> 02:16:56,440 AND WHO IS GETTING ACCESS TO 3527 02:16:56,440 --> 02:16:58,160 CLINICAL TRIALS SO WE'RE NOT 3528 02:16:58,160 --> 02:16:59,800 ABLE TO SAY MEANINGFUL CLINICAL 3529 02:16:59,800 --> 02:17:00,480 AND ACTIONABLE RESULTS FROM 3530 02:17:00,480 --> 02:17:02,520 CLINICAL TRIALS IF WE DON'T HAVE 3531 02:17:02,520 --> 02:17:03,600 A DIVERSE PATIENT POPULATION 3532 02:17:03,600 --> 02:17:05,800 ENROLLED IN THOSE TRIALS IN THE 3533 02:17:05,800 --> 02:17:07,000 FIRST PLACE. 3534 02:17:07,000 --> 02:17:08,760 THE INFORMED CONSENT PROCESS IS 3535 02:17:08,760 --> 02:17:12,720 OFTEN THE TIME POINT AT WHICH 3536 02:17:12,720 --> 02:17:13,920 MANY ELIGIBLE FAMILIES DON'T 3537 02:17:13,920 --> 02:17:16,760 FOLLOW THROUGH AND WE KNOW THIS 3538 02:17:16,760 --> 02:17:18,880 BECAUSE THERE ARE -- THERE'S 3539 02:17:18,880 --> 02:17:20,160 ABJECT -- SOMETIMES MISTRUST OF 3540 02:17:20,160 --> 02:17:22,480 THE HEALTH CARE SYSTEM, EVEN 3541 02:17:22,480 --> 02:17:24,000 MORE MISTRUST OF THE RESEARCH 3542 02:17:24,000 --> 02:17:31,440 ENTERPRISE, SO IT WAS MENTIONED 3543 02:17:31,440 --> 02:17:32,360 EARLIER INDIGENOUS AND NATIVE 3544 02:17:32,360 --> 02:17:34,040 COMMUNITIES ARE AT THE TOP OF 3545 02:17:34,040 --> 02:17:36,400 THE LIST DIRECTLY HARMED BY 3546 02:17:36,400 --> 02:17:37,880 RESEARCH IN THE PASS. 3547 02:17:37,880 --> 02:17:41,520 THERE'S A CULTURAL COMPETENCY 3548 02:17:41,520 --> 02:17:44,640 THAT HAS TO BE EMBEDDED IN THE 3549 02:17:44,640 --> 02:17:45,760 INFORMED CONSENT PROCESS, VERY 3550 02:17:45,760 --> 02:17:47,200 PATIENT AND FAMILY SPECIFIC, 3551 02:17:47,200 --> 02:17:51,040 REQUIRES A LOT MORE THAN SIMPLY 3552 02:17:51,040 --> 02:17:52,320 UNDERSTANDING THE RISK. 3553 02:17:52,320 --> 02:17:59,040 UNDERSTANDING AND COMMUNICATING 3554 02:17:59,040 --> 02:18:03,360 RISKS AND BENEFITS OF THE 3555 02:18:03,360 --> 02:18:03,720 INTERVENTIONS. 3556 02:18:03,720 --> 02:18:05,520 >> I WANTED TO SEE IF WE HAVE 3557 02:18:05,520 --> 02:18:16,040 QUESTIONS FROM THE AUDIENCE. 3558 02:18:16,400 --> 02:18:17,400 3559 02:18:17,400 --> 02:18:21,320 >> WORRY TALKING ABOUT INFORMED 3560 02:18:21,320 --> 02:18:21,920 CONSENT -- WE'RE TALKING ABOUT 3561 02:18:21,920 --> 02:18:23,280 INFORMED CONSENT. 3562 02:18:23,280 --> 02:18:25,520 MANY OF YOU KNOW I WORK IN THE 3563 02:18:25,520 --> 02:18:27,840 RARE DISEASE SPACE. 3564 02:18:27,840 --> 02:18:30,880 A CHALLENGE THAT I SEE, WE 3565 02:18:30,880 --> 02:18:33,840 AREN'T TO A CLINICAL TRIAL YET 3566 02:18:33,840 --> 02:18:35,360 BUT I'VE OBSERVED MANY PATIENT 3567 02:18:35,360 --> 02:18:38,400 GROUPS THAT GO THROUGH THIS 3568 02:18:38,400 --> 02:18:39,960 BECAUSE FAMILIES ARE SO 3569 02:18:39,960 --> 02:18:41,600 DESPERATE, ESPECIALLY IN THE 3570 02:18:41,600 --> 02:18:46,640 RARE DISEASE SPACE, LOOKING FOR 3571 02:18:46,640 --> 02:18:47,520 A TREATMENT. 3572 02:18:47,520 --> 02:18:50,080 I FIND A GAP BEFORE INFORMED 3573 02:18:50,080 --> 02:18:56,120 CONSENT OF GETTING AN EDUCATION 3574 02:18:56,120 --> 02:18:58,760 OR BASIC SIMPLE MESSAGE OUT TO 3575 02:18:58,760 --> 02:19:02,120 INCLUSION AND EXCLUSION CRITERIA 3576 02:19:02,120 --> 02:19:03,760 BECAUSE WHAT I'M SEEING, EVEN 3577 02:19:03,760 --> 02:19:05,880 TRYING TO EDUCATE OUR OWN 3578 02:19:05,880 --> 02:19:07,600 COMMUNITY IS UNDERSTANDING THAT 3579 02:19:07,600 --> 02:19:13,080 NOT EVERYONE WILL BE ABLE TO 3580 02:19:13,080 --> 02:19:13,640 PARTICIPATE. 3581 02:19:13,640 --> 02:19:15,800 THEN THAT INADVERTENTLY CAUSES A 3582 02:19:15,800 --> 02:19:23,640 DIVISION WITHIN THE GROUP 3583 02:19:23,640 --> 02:19:24,040 ITSELF. 3584 02:19:24,040 --> 02:19:25,880 I DON'T KNOW MANY THAT HAD THIS 3585 02:19:25,880 --> 02:19:27,520 HAPPEN BUT I SEE THERE'S A GAP. 3586 02:19:27,520 --> 02:19:29,160 THIS IS THE TEACHER COMING OUT 3587 02:19:29,160 --> 02:19:30,920 IN ME. 3588 02:19:30,920 --> 02:19:33,960 HOW DO WE EDUCATE THESE FAMILIES 3589 02:19:33,960 --> 02:19:42,840 IN A GINGER WAY, BECAUSE THIS IS 3590 02:19:42,840 --> 02:19:43,720 ALL HIGHLY SENSITIVE. 3591 02:19:43,720 --> 02:19:44,760 WE ALL WANT TREATMENT. 3592 02:19:44,760 --> 02:19:46,560 BUT HOW DO WE COME ACROSS BEFORE 3593 02:19:46,560 --> 02:19:49,520 WE GET TO THE INFORMED CONSENT 3594 02:19:49,520 --> 02:19:51,400 EDUCATING THE PATIENT COMMUNITY 3595 02:19:51,400 --> 02:19:54,320 ON HOW WE GET TO EVEN AN 3596 02:19:54,320 --> 02:19:56,080 INFORMED CONSENT FOR THESE 3597 02:19:56,080 --> 02:19:58,320 THINGS AND UNDERSTANDING THE 3598 02:19:58,320 --> 02:20:03,680 RISKS. 3599 02:20:03,680 --> 02:20:05,320 DOES THAT MAKE SENSE? 3600 02:20:05,320 --> 02:20:06,520 >> THIS IS REALLY HARD. 3601 02:20:06,520 --> 02:20:09,920 THIS IS THE HARDEST THING I DO. 3602 02:20:09,920 --> 02:20:14,080 A COUPLE MONTHS AGO ACTUALLY A 3603 02:20:14,080 --> 02:20:17,000 MOTHER CALLED ME AND SAID I WANT 3604 02:20:17,000 --> 02:20:17,800 TO START A LETTER-WRITING 3605 02:20:17,800 --> 02:20:19,760 CAMPAIGN TO A COMPANY TO PUT 3606 02:20:19,760 --> 02:20:25,120 PRESSURE ON THEM TO OPEN A 3607 02:20:25,120 --> 02:20:26,000 CLINICAL TRIAL. 3608 02:20:26,000 --> 02:20:31,800 AND SHE WANTED ME TO HELP HER 3609 02:20:31,800 --> 02:20:34,000 LEVERAGE FUNDING FOR THIS. 3610 02:20:34,000 --> 02:20:35,880 SHE'S VERY PASSIONATE AND 3611 02:20:35,880 --> 02:20:38,280 DESPERATE AND I SAID -- SHE HAD 3612 02:20:38,280 --> 02:20:40,520 A CHILD WITH ADVANCED MLD. 3613 02:20:40,520 --> 02:20:43,800 IT WAS CLEAR SHE THOUGHT IF SHE 3614 02:20:43,800 --> 02:20:45,680 DID THIS THE CHILD WOULD BE IN 3615 02:20:45,680 --> 02:20:46,360 THE TRIAL. 3616 02:20:46,360 --> 02:20:48,520 I WENT RIGHT IN BECAUSE THAT'S 3617 02:20:48,520 --> 02:20:48,960 HOW I AM. 3618 02:20:48,960 --> 02:20:51,280 I SAID THIS IS ALL WONDERFUL AND 3619 02:20:51,280 --> 02:20:52,280 INTRIGUING AND INTERESTING. 3620 02:20:52,280 --> 02:20:57,280 I HAVEN'T HEARD ANYTHING ABOUT 3621 02:20:57,280 --> 02:20:59,120 ANY OF THIS. 3622 02:20:59,120 --> 02:21:09,600 HOWEVER -- I DON'T SWAN NOT 3623 02:22:10,160 --> 02:22:13,400 SERVING THE GREATER PURPOSE OF 3624 02:22:13,400 --> 02:22:13,920 THE RESEARCH. 3625 02:22:13,920 --> 02:22:17,400 THE SECOND THING I SAY IS THAT, 3626 02:22:17,400 --> 02:22:23,120 YOU KNOW, THIS IS NOT ABOUT YOUR 3627 02:22:23,120 --> 02:22:24,400 CHILD. 3628 02:22:24,400 --> 02:22:26,080 THIS IS ABOUT SCIENCE, 3629 02:22:26,080 --> 02:22:33,080 MEASUREMENTS, DATA COLLECTION. 3630 02:22:33,080 --> 02:22:40,600 THIS MAKES PEOPLE ANTSY. 3631 02:22:40,600 --> 02:22:41,680 IF PAUL ORCHARD TREATED EVERY 3632 02:22:41,680 --> 02:22:43,120 KID HE WOULD NOT HAVE MEANINGFUL 3633 02:22:43,120 --> 02:22:44,960 DATA TO SHOW THE FDA. 3634 02:22:44,960 --> 02:22:47,720 ALL THIS WORK WOULD BE FOR 3635 02:22:47,720 --> 02:22:47,960 NAUGHT. 3636 02:22:47,960 --> 02:22:50,040 I EXPLAINED THAT TO THE 3637 02:22:50,040 --> 02:22:51,720 COMMUNITY ON AS GENTLY AND 3638 02:22:51,720 --> 02:22:54,840 CALMLY AND RATIONALLY AS I CAN. 3639 02:22:54,840 --> 02:22:57,360 AGAIN, WITH THE TEFLON, KEVLAR 3640 02:22:57,360 --> 02:23:01,960 CHILD OF MY DAUGHTER COULD NEVER 3641 02:23:01,960 --> 02:23:02,200 BENEFIT. 3642 02:23:02,200 --> 02:23:04,600 I SAY DON'T BECOME A PATIENT 3643 02:23:04,600 --> 02:23:07,000 ADVOCATE IF YOU CAN'T ACCEPT THE 3644 02:23:07,000 --> 02:23:08,520 FACT YOUR KID WON'T BENEFIT. 3645 02:23:08,520 --> 02:23:09,720 IT'S A CONTROVERSIAL THING TO 3646 02:23:09,720 --> 02:23:11,480 SAY, SOME BEST WORK HAS BEEN 3647 02:23:11,480 --> 02:23:12,800 DONE BY PARENTS FOR THEIR 3648 02:23:12,800 --> 02:23:14,440 CHILDREN BUT IF YOU THINK THIS 3649 02:23:14,440 --> 02:23:17,280 IS THE ONLY OUTCOME THAT YOU 3650 02:23:17,280 --> 02:23:18,600 WANT, THEN DO SOMETHING ELSE 3651 02:23:18,600 --> 02:23:19,720 WITH YOUR TIME. 3652 02:23:19,720 --> 02:23:23,200 IT'S NOT GOOD FOR THE COMMUNITY 3653 02:23:23,200 --> 02:23:24,440 OR RESEARCH. 3654 02:23:24,440 --> 02:23:25,720 THAT'S MY EXPERIENCE, TEN YEARS 3655 02:23:25,720 --> 02:23:29,200 INTO THIS SPACE. 3656 02:23:29,200 --> 02:23:38,960 3657 02:23:38,960 --> 02:23:41,200 >> IT GOES BACK TO EARLIER 3658 02:23:41,200 --> 02:23:42,160 CONVERSATIONS, HAVE TO BEGIN 3659 02:23:42,160 --> 02:23:45,560 WITH PATIENT AND FAMILY 3660 02:23:45,560 --> 02:23:45,880 ORGANIZATIONS. 3661 02:23:45,880 --> 02:23:47,200 WHETHER INDUSTRY OR ACADEMIC, 3662 02:23:47,200 --> 02:23:48,960 HAS TO LISTEN TO WHAT'S 3663 02:23:48,960 --> 02:23:52,960 IMPORTANT TO THOSE FAMILIES. 3664 02:23:52,960 --> 02:23:53,760 THAT'S ESSENTIAL. 3665 02:23:53,760 --> 02:23:56,160 OUTCOME MEASURES ARE EQUALLY AS 3666 02:23:56,160 --> 02:23:56,440 IMPORTANT. 3667 02:23:56,440 --> 02:23:58,760 WE LOOK FOR BIOMARKERS BUT IN 3668 02:23:58,760 --> 02:24:00,400 ALL OF THAT, HAS TO BE CAREFULLY 3669 02:24:00,400 --> 02:24:05,360 LAID OUT ON BEHALF OF ADVOCACY 3670 02:24:05,360 --> 02:24:06,880 ORGANIZATIONS TO FAMILIES GENTLY 3671 02:24:06,880 --> 02:24:09,600 BUT DISCUSSIONS OF CRITERIA HAS 3672 02:24:09,600 --> 02:24:11,720 TO HAPPEN WITH INVESTIGATORS, 3673 02:24:11,720 --> 02:24:13,200 CLINICAL TEAM AND FAMILIES. 3674 02:24:13,200 --> 02:24:14,760 ONE STUDY, THE INDUSTRY HAD 3675 02:24:14,760 --> 02:24:15,400 STAFF MEMBERS TALKING TO 3676 02:24:15,400 --> 02:24:16,600 FAMILIES SAYING YOU'RE NOT 3677 02:24:16,600 --> 02:24:17,000 ELIGIBLE. 3678 02:24:17,000 --> 02:24:18,360 THAT IS NOT APPROPRIATE. 3679 02:24:18,360 --> 02:24:20,040 IT HAS TO COME FROM THE 3680 02:24:20,040 --> 02:24:21,320 CLINICIAN WHO KNOWS MEDICAL DATA 3681 02:24:21,320 --> 02:24:23,960 TO CAREFULLY AND GENTLY DISCUSS 3682 02:24:23,960 --> 02:24:26,360 THIS AND HAS TO BE ALSO 3683 02:24:26,360 --> 02:24:30,280 REALLY -- I LIKE THE WORD "CARE" 3684 02:24:30,280 --> 02:24:32,920 MORE THAN "CURE." 3685 02:24:32,920 --> 02:24:35,760 I WOULD SAY THEY ARE THE SAME 3686 02:24:35,760 --> 02:24:37,080 WORD, ONE LETTER DIFFERENT, 3687 02:24:37,080 --> 02:24:37,640 EQUALLY IMPORTANT. 3688 02:24:37,640 --> 02:24:39,960 WHEN I DISCUSS WHY A CHILD NOT 3689 02:24:39,960 --> 02:24:42,120 BE ELIGIBLE, I PIVOT AND DISCUSS 3690 02:24:42,120 --> 02:24:43,880 THINGS TO OPTIMIZE THAT CHILD'S 3691 02:24:43,880 --> 02:24:45,320 CARE, EQUALLY AS IMPORTANT FOR 3692 02:24:45,320 --> 02:24:47,920 THE NEXT STUDY COMING DOWN THE 3693 02:24:47,920 --> 02:24:51,400 PIPELINE, WE NEED THAT CHILD 3694 02:24:51,400 --> 02:24:52,440 OPTIMIZE THEIR CARE. 3695 02:24:52,440 --> 02:24:56,440 THAT'S A WHICH TO DEAL WITH A 3696 02:24:56,440 --> 02:24:59,520 DIFFICULT ISSUE, IT HAS TO BE A 3697 02:24:59,520 --> 02:25:01,080 DELICATE DISCUSSION BETWEEN 3698 02:25:01,080 --> 02:25:01,760 CLINICIAN AND FAMILIES. 3699 02:25:01,760 --> 02:25:04,320 >> DID YOU HAVE ANY COMMENTS ON 3700 02:25:04,320 --> 02:25:04,520 THIS? 3701 02:25:04,520 --> 02:25:14,960 >> THOSE ARE ALL REALLY 3702 02:25:20,880 --> 02:25:24,080 CRITICAL, REMINDING ME OF A FILM 3703 02:25:24,080 --> 02:25:25,040 CALLED "RARE" ABOUT -- I DON'T 3704 02:25:25,040 --> 02:25:26,920 KNOW IF PEOPLE HAVE SEEN IT IN 3705 02:25:26,920 --> 02:25:28,880 THIS ROOM BUT I RECOMMEND IT. 3706 02:25:28,880 --> 02:25:38,920 IT'S ABOUT A MOTHER AND A 3707 02:25:38,920 --> 02:25:42,920 DAUGHTER DUO, ABOUT THEIR 3708 02:25:42,920 --> 02:25:47,480 DIAGNOSTICS AND TREATMENT 3709 02:25:47,480 --> 02:25:49,440 JOURNEY WITH HERMANSKI PODLOX 3710 02:25:49,440 --> 02:25:52,320 SYNDROME, A SIMILAR STORY, CAME 3711 02:25:52,320 --> 02:26:01,480 TO THE NIH, NHGRI, HUMAN GENOME 3712 02:26:01,480 --> 02:26:02,480 RESEARCH INSTITUTE, THEY HAD 3713 02:26:02,480 --> 02:26:04,440 FOUND A SET OF INVESTIGATORS 3714 02:26:04,440 --> 02:26:06,400 THAT COULD TRANSLATE SOME BASIC 3715 02:26:06,400 --> 02:26:08,880 SCIENCE RESEARCH AND MOVE IT 3716 02:26:08,880 --> 02:26:10,880 INTO PHASE 1 AND 2 CLINICAL 3717 02:26:10,880 --> 02:26:13,880 TRIALS, AND HER OWN DAUGHTER WAS 3718 02:26:13,880 --> 02:26:16,680 INELIGIBLE FOR THE STUDY THAT 3719 02:26:16,680 --> 02:26:18,200 THEY EVENTUALLY GOT OFF THE 3720 02:26:18,200 --> 02:26:20,640 GROUND AND THERE'S LIKE THIS 3721 02:26:20,640 --> 02:26:21,760 REALLY HEART WRENCHING SCENE IN 3722 02:26:21,760 --> 02:26:26,200 WHICH HERE IS A MOM WHO HAS DONE 3723 02:26:26,200 --> 02:26:30,920 EVERYTHING, HAD THE BAKE SALES, 3724 02:26:30,920 --> 02:26:32,680 STARTED THESE FOUNDATIONS, 3725 02:26:32,680 --> 02:26:34,040 ACTUALLY GATHERED ALL OF THE 30 3726 02:26:34,040 --> 02:26:35,720 OF 30 PATIENTS IN THE UNITED 3727 02:26:35,720 --> 02:26:38,360 STATES THAT HAS THIS RARE 3728 02:26:38,360 --> 02:26:42,520 SYNDROME VIA SOCIAL MEDIA AND 3729 02:26:42,520 --> 02:26:43,280 OTHER RESOURCE CONNECTIONS AND 3730 02:26:43,280 --> 02:26:44,680 HER OWN DAUGHTER IS NOT ABLE TO 3731 02:26:44,680 --> 02:26:46,760 BE IN THE TRIAL BASED ON 3732 02:26:46,760 --> 02:26:48,640 INCLUSION AND EXCLUSION CRITERIA 3733 02:26:48,640 --> 02:26:54,120 SO I THINK THAT NOT TO ADDRESS 3734 02:26:54,120 --> 02:26:55,440 YOUR QUESTION WITH ACTIONABLE 3735 02:26:55,440 --> 02:26:58,040 ITEMS, RATHER THIS IS TO HAVE 3736 02:26:58,040 --> 02:26:59,040 RESEARCHERS REALLY UNDERSTAND 3737 02:26:59,040 --> 02:27:02,400 THE IMPACT THAT HAS ON FAMILIES 3738 02:27:02,400 --> 02:27:06,480 AND TO I THINK SEEING THAT KIND 3739 02:27:06,480 --> 02:27:09,320 OF ON FILM IT BRINGS THE EMOTION 3740 02:27:09,320 --> 02:27:11,240 THAT HAS TO HAPPEN WHEN YOU HAVE 3741 02:27:11,240 --> 02:27:13,840 THOSE CONVERSATIONS. 3742 02:27:13,840 --> 02:27:14,080 3743 02:27:14,080 --> 02:27:24,600 >> AND IS A TOPIC FREQUENTLY IN 3744 02:27:25,400 --> 02:27:27,760 MY JOB, RDCRN AND COALITION OF 3745 02:27:27,760 --> 02:27:28,840 PATIENT ADVOCATES THIS HAS COME 3746 02:27:28,840 --> 02:27:30,040 UP IN OUR DISCUSSION. 3747 02:27:30,040 --> 02:27:31,240 ARE THERE OTHER QUESTIONS IN THE 3748 02:27:31,240 --> 02:27:32,960 AUDIENCE? 3749 02:27:32,960 --> 02:27:35,760 OR ONLINE? 3750 02:27:35,760 --> 02:27:36,960 3751 02:27:36,960 --> 02:27:38,920 MY NEXT QUESTION FOR THE GROUP 3752 02:27:38,920 --> 02:27:41,520 THEN IS WHAT CHANGES DO YOU SEE 3753 02:27:41,520 --> 02:27:44,840 IN THE NEXT FIVE YEARS THAT ARE 3754 02:27:44,840 --> 02:27:47,680 GOING TO DRASTICALLY CHANGE THE 3755 02:27:47,680 --> 02:27:48,200 FIELD? 3756 02:27:48,200 --> 02:27:53,040 WHO WOULD LIKE TO START? 3757 02:27:53,040 --> 02:27:54,440 >> SURE. 3758 02:27:54,440 --> 02:27:55,400 SO, MAYBE UNSURPRISINGLY, I 3759 02:27:55,400 --> 02:28:00,040 SPENT A LOT OF THE LAST PROBABLY 3760 02:28:00,040 --> 02:28:03,960 TWO TO THREE YEARS ON THE 3761 02:28:03,960 --> 02:28:04,840 CLINICAL INTEGRATION OF 3762 02:28:04,840 --> 02:28:06,280 ARTIFICIAL INTELLIGENCE ACROSS 3763 02:28:06,280 --> 02:28:08,680 RESEARCH AND CLINICAL CARE. 3764 02:28:08,680 --> 02:28:11,320 AND ONE OF THE I THINK REALLY 3765 02:28:11,320 --> 02:28:13,960 EXCITING ADVANCES IN THIS AREA 3766 02:28:13,960 --> 02:28:16,440 IS WE MOVE AND FURTHER 3767 02:28:16,440 --> 02:28:19,440 SOPHISTICATE A.I. INTO CLINICAL 3768 02:28:19,440 --> 02:28:21,480 RESEARCH REALMS IS THE NEW IDEA 3769 02:28:21,480 --> 02:28:24,560 OF A DIGITAL TWIN. 3770 02:28:24,560 --> 02:28:27,600 AND DIGITAL TWINS ARE 3771 02:28:27,600 --> 02:28:31,000 ESSENTIALLY MULTI-MODAL REALLY 3772 02:28:31,000 --> 02:28:38,760 ADVANCED MULTI-MODAL DATASETS OF 3773 02:28:38,760 --> 02:28:42,720 EHR RECORD, EMERGING E HR WITH 3774 02:28:42,720 --> 02:28:44,160 WEARABLE WITH EXOME SEQUENCING, 3775 02:28:44,160 --> 02:28:44,920 CREATING A DIGITAL 3776 02:28:44,920 --> 02:28:46,120 REPRESENTATION OF THAT PERSON 3777 02:28:46,120 --> 02:28:50,040 WHICH CAN BE A STAND-IN FOR 3778 02:28:50,040 --> 02:28:52,360 FIRST-IN-HUMAN CLINICAL TRIALS 3779 02:28:52,360 --> 02:28:56,640 RESEARCH AS SOME SORT OF INITIAL 3780 02:28:56,640 --> 02:28:58,600 BEFORE PHASE 1 IMPOSED 3781 02:28:58,600 --> 02:29:00,240 PRE-CLINICAL AND IN ANIMAL 3782 02:29:00,240 --> 02:29:00,560 STUDIES. 3783 02:29:00,560 --> 02:29:04,480 WHAT'S REALLY EXCITING ABOUT 3784 02:29:04,480 --> 02:29:05,920 THIS, THERE'S STILL A LOT OF 3785 02:29:05,920 --> 02:29:07,440 WORK AND CONTROVERSY, IT REALLY 3786 02:29:07,440 --> 02:29:09,760 ADDRESSES SOME OF THE VERY ACUTE 3787 02:29:09,760 --> 02:29:14,920 ETHICAL CONCERNS WE HAVE IN 3788 02:29:14,920 --> 02:29:18,320 THOSE PHASE 1 TRIALS AND WE'RE 3789 02:29:18,320 --> 02:29:20,640 LEVERAGING MASSIVE DATASETS, 3790 02:29:20,640 --> 02:29:29,360 ABILITY TO SYSTEMATICALLY 3791 02:29:29,360 --> 02:29:31,480 ANALYZE AND ACROSS ANGLES AND 3792 02:29:31,480 --> 02:29:31,840 EXPOSURES. 3793 02:29:31,840 --> 02:29:33,200 THIS IS I THINK GOING TO BE 3794 02:29:33,200 --> 02:29:36,920 FURTHER ADVANCED IN THE NEXT 3795 02:29:36,920 --> 02:29:38,440 FIVE YEARS, TO WHAT POINT THEY 3796 02:29:38,440 --> 02:29:40,640 CAN BE AN ACTUAL STAND-IN TO 3797 02:29:40,640 --> 02:29:42,360 CLINICAL TRIALS IS AN 3798 02:29:42,360 --> 02:29:45,720 UNANSWERABLE QUESTION TODAY BUT 3799 02:29:45,720 --> 02:29:50,720 I AM REALLY ENCOURAGED BY JUST 3800 02:29:50,720 --> 02:29:52,240 THE AMAZING TECHNOLOGIES WE CAN 3801 02:29:52,240 --> 02:29:54,640 LEVERAGE IN MEDICAL A.I. AND 3802 02:29:54,640 --> 02:29:56,200 WORKED ALONGSIDE ADDRESSING SOME 3803 02:29:56,200 --> 02:29:57,840 OF THE ACUTE ETHICAL ISSUES. 3804 02:29:57,840 --> 02:30:00,920 SO CERTAINLY IN DATA PRIVACY AND 3805 02:30:00,920 --> 02:30:02,440 PROTECTION WE RECENTLY PUBLISHED 3806 02:30:02,440 --> 02:30:07,040 A PAPER A HOW A DIGITAL TWIN 3807 02:30:07,040 --> 02:30:08,360 COULD ACCOUNT FOR SOCIAL 3808 02:30:08,360 --> 02:30:11,080 DETERMINANTS OF HEALTH, SO WE 3809 02:30:11,080 --> 02:30:14,040 KNOW VERY LITTLE ABOUT 3810 02:30:14,040 --> 02:30:15,160 ENVIRONMENTAL HEALTH EXPOSURES, 3811 02:30:15,160 --> 02:30:17,360 WE KNOW THAT EDUCATION AND 3812 02:30:17,360 --> 02:30:21,720 INCOME AND OTHER SOCIAL FACTORS 3813 02:30:21,720 --> 02:30:23,120 HAVE VERY SIGNIFICANT IMPACT ON 3814 02:30:23,120 --> 02:30:24,120 PEOPLE'S HEALTH STATUS OVER THE 3815 02:30:24,120 --> 02:30:26,640 LIFE COURSE. 3816 02:30:26,640 --> 02:30:31,440 BUT MODELING THAT IN A POTENTIAL 3817 02:30:31,440 --> 02:30:35,320 A.I. TWIN AND USING THAT 3818 02:30:35,320 --> 02:30:36,280 REPRESENTATION IN VERY RISKY 3819 02:30:36,280 --> 02:30:39,480 TRIALS AT THE VERY EARLY STAGES 3820 02:30:39,480 --> 02:30:41,640 OF DRUG DEVELOPMENT WILL NEED A 3821 02:30:41,640 --> 02:30:45,760 LOT OF REFINING BUT THAT'S ONE 3822 02:30:45,760 --> 02:30:47,400 AREA WE'RE PAYING CLOSE 3823 02:30:47,400 --> 02:30:48,800 ATTENTION TO. 3824 02:30:48,800 --> 02:30:50,320 >> I WOULD EXTEND THIS WITH WHAT 3825 02:30:50,320 --> 02:30:53,080 YOU SAID ABOUT A.I., I WAS 3826 02:30:53,080 --> 02:30:55,720 ASKING A DISCUSSION WITH MARIA 3827 02:30:55,720 --> 02:30:58,760 BEFORE ABOUT SOMETIMES THERE'S A 3828 02:30:58,760 --> 02:31:01,280 REQUIREMENT FOR A PLACEBO 3829 02:31:01,280 --> 02:31:04,360 CONTROL GROUP AND MANY DISEASES 3830 02:31:04,360 --> 02:31:05,440 ARE DEVASTATING. 3831 02:31:05,440 --> 02:31:08,000 AND SO I THINK, MARIA, IF YOU 3832 02:31:08,000 --> 02:31:10,240 DON'T MIND EXTENDING THE 3833 02:31:10,240 --> 02:31:11,560 QUESTION ABOUT THE CONCERNS 3834 02:31:11,560 --> 02:31:13,640 ABOUT PLACEBO CONTROL IN THESE 3835 02:31:13,640 --> 02:31:15,160 TRIALS AND THEN IF ANYBODY WANTS 3836 02:31:15,160 --> 02:31:17,480 TO TALK ABOUT THE NEED FOR 3837 02:31:17,480 --> 02:31:17,720 PLACEBO. 3838 02:31:17,720 --> 02:31:21,080 >> THIS IS SO CRITICAL. 3839 02:31:21,080 --> 02:31:25,160 I FEEL LIKE -- I WANT TO HUG 3840 02:31:25,160 --> 02:31:26,800 EVERYBODY HERE BECAUSE WE 3841 02:31:26,800 --> 02:31:28,560 WOULDN'T BE THIS CLOSE HAD IT 3842 02:31:28,560 --> 02:31:30,840 NOT BEEN FOR THE NIH. 3843 02:31:30,840 --> 02:31:39,080 AND THEIR WORK IN NATURAL 3844 02:31:39,080 --> 02:31:39,640 HISTORY. 3845 02:31:39,640 --> 02:31:42,680 AND TINA ERB, WHEREVER YOU ARE, 3846 02:31:42,680 --> 02:31:44,080 RESPECT, LOVE. 3847 02:31:44,080 --> 02:31:47,640 TINA ERB, OUR PROGRAM OFFICER AT 3848 02:31:47,640 --> 02:31:50,240 THE RDCRN HAS SCHOOLED ME FOR 3849 02:31:50,240 --> 02:31:52,000 SEVERAL YEARS ON THE CRITICAL 3850 02:31:52,000 --> 02:31:56,720 ROLE OF NATURAL HISTORY DATA 3851 02:31:56,720 --> 02:31:57,800 COLLECTION. 3852 02:31:57,800 --> 02:32:00,000 AND DATA IS POLITICAL. 3853 02:32:00,000 --> 02:32:01,520 IT IS NOT OBJECTIVE. 3854 02:32:01,520 --> 02:32:03,400 IT IS POLITICAL. 3855 02:32:03,400 --> 02:32:06,640 AND THE FDA HAS A TREMENDOUS 3856 02:32:06,640 --> 02:32:09,640 CHALLENGE, WHICH IS THESE GENE 3857 02:32:09,640 --> 02:32:11,240 THERAPIES ARE COMING THROUGH, 3858 02:32:11,240 --> 02:32:12,440 VERY EXPENSIVE, NO ONE KNOW WHAT 3859 02:32:12,440 --> 02:32:13,680 IS THEY ARE. 3860 02:32:13,680 --> 02:32:16,720 THEY ARE PRETTY SCARY WHEN YOU 3861 02:32:16,720 --> 02:32:23,160 THINK ABOUT IT. 3862 02:32:23,160 --> 02:32:25,360 LENTIVIRAL VECTORS, WE DID A 3863 02:32:25,360 --> 02:32:27,440 PIECE AND I GOT CRAZY LETTERS. 3864 02:32:27,440 --> 02:32:29,400 I THOUGHT IT WAS AMAZING. 3865 02:32:29,400 --> 02:32:31,480 OTHERS THOUGHT IT WAS A 3866 02:32:31,480 --> 02:32:32,280 CONSPIRACY, BILL GATES COMING 3867 02:32:32,280 --> 02:32:34,160 FOR US OR SOMETHING. 3868 02:32:34,160 --> 02:32:36,440 IN ALL SERIOUSNESS, WE'RE IN A 3869 02:32:36,440 --> 02:32:39,880 SITUATION WHERE WE HAVE TO 3870 02:32:39,880 --> 02:32:41,120 ENGAGE WITH REGULATORS AS SOON 3871 02:32:41,120 --> 02:32:42,360 AS WE'RE THINKING OF A CLINICAL 3872 02:32:42,360 --> 02:32:43,640 TRIAL TO GET THEM COMFORTABLE 3873 02:32:43,640 --> 02:32:47,280 WITH THE DATA WE'RE GOING TO 3874 02:32:47,280 --> 02:32:47,480 HAVE. 3875 02:32:47,480 --> 02:32:50,320 AND THAT'S NOT -- AND I DON'T 3876 02:32:50,320 --> 02:32:55,080 BELIEVE IN WEAPONIZING HUMAN 3877 02:32:55,080 --> 02:32:57,760 EMOTIONS WITH THE FDA. 3878 02:32:57,760 --> 02:33:01,960 WE OWE A TREMENDOUS DEBT TO AIDS 3879 02:33:01,960 --> 02:33:06,200 ACTIVISM OF THE 1980s, 3880 02:33:06,200 --> 02:33:07,200 REVOLUTIONARY, BUT THROWING THE 3881 02:33:07,200 --> 02:33:09,800 ASHES OF YOUR LOVED ONE ON THE 3882 02:33:09,800 --> 02:33:10,800 WHITE HOUSE LAWN PROBABLY WON'T 3883 02:33:10,800 --> 02:33:14,640 GET YOU ANY WHERE THESE DAYS. 3884 02:33:14,640 --> 02:33:20,000 YOU NEED TO ENGAGE WITH FDA, USE 3885 02:33:20,000 --> 02:33:23,480 TOOLS OF NIH TO BE COMFORTABLE 3886 02:33:23,480 --> 02:33:24,800 SIGNING OFF A MULTI-MILLION 3887 02:33:24,800 --> 02:33:25,560 DOLLAR THERAPY THAT DOESN'T 3888 02:33:25,560 --> 02:33:31,040 NECESSARILY HAVE A HUGE DATASET. 3889 02:33:31,040 --> 02:33:32,680 OUR CLINICIANS INCLUDING THE 3890 02:33:32,680 --> 02:33:34,960 AMAZING PAUL ORCHARD WENT TO THE 3891 02:33:34,960 --> 02:33:39,440 FDA AND MADE IT CLEAR SOME 3892 02:33:39,440 --> 02:33:41,320 THINGS THEY WERE ASKING WERE 3893 02:33:41,320 --> 02:33:42,200 IMPOSSIBLE, IN PARTICULAR 3894 02:33:42,200 --> 02:33:46,600 PLACEBO CONTROL WITH A BONE 3895 02:33:46,600 --> 02:33:48,440 MARROW TRANSPLANT, FOR MLD 3896 02:33:48,440 --> 02:33:49,440 CHILDREN FACING 30% MORTALITY 3897 02:33:49,440 --> 02:33:52,360 RATE AND WE COULD NOT SIGN UP ON 3898 02:33:52,360 --> 02:33:54,000 A PLACEBO CONTROL ARM THAT WOULD 3899 02:33:54,000 --> 02:33:56,640 KILL 30% OF THE BABIES. 3900 02:33:56,640 --> 02:33:58,160 THAT DISCUSSION WAS 3901 02:33:58,160 --> 02:33:59,720 UNCOMFORTABLE FOR THE ACADEMIC 3902 02:33:59,720 --> 02:33:59,920 TEAMS. 3903 02:33:59,920 --> 02:34:01,920 WE DON'T KNOW WHAT WE DON'T 3904 02:34:01,920 --> 02:34:02,120 KNOW. 3905 02:34:02,120 --> 02:34:04,400 THEY LIKE TO HEDGE THEIR BETS. 3906 02:34:04,400 --> 02:34:08,760 WE HAD TO MAKE OUR CLINICAL 3907 02:34:08,760 --> 02:34:10,200 TEAMS LISTEN, SAY I DON'T WANT 3908 02:34:10,200 --> 02:34:11,840 TO USE THIS PLACEBO CONTROL ARM. 3909 02:34:11,840 --> 02:34:19,040 I KNOW YOU NEED IT TO COVER YOUR 3910 02:34:19,040 --> 02:34:20,600 FLANK, PART OF YOUR ANATOMY, YOU 3911 02:34:20,600 --> 02:34:23,120 DON'T WANT TO BE TAKEN OUT BY 3912 02:34:23,120 --> 02:34:24,200 THE "NEW YORK TIMES," RIGHT? 3913 02:34:24,200 --> 02:34:25,200 I GET THAT. 3914 02:34:25,200 --> 02:34:27,120 AT THE SAME TIME WE CAN'T SIGN 3915 02:34:27,120 --> 02:34:27,920 OFF. 3916 02:34:27,920 --> 02:34:31,520 YOU HAVE TO HAVE REALLY POINTED 3917 02:34:31,520 --> 02:34:31,880 CONVERSATIONS. 3918 02:34:31,880 --> 02:34:38,280 THE NIH IS SWITZERLAND, GIVING 3919 02:34:38,280 --> 02:34:41,520 THEM TO IS A DISCUSSION NOT 3920 02:34:41,520 --> 02:34:44,800 DRIVEN PURELY BY DESPERATE 3921 02:34:44,800 --> 02:34:51,720 PARENTS OR BUSINESS INTERESTS 3922 02:34:51,720 --> 02:34:52,880 LOOKING AT RUNWAYS RUNNING OUT 3923 02:34:52,880 --> 02:34:54,840 OF MONEY, WE'RE GOING TO GIVE 3924 02:34:54,840 --> 02:34:57,600 YOU EVERYTHING BUT WILL MOSTLY 3925 02:34:57,600 --> 02:35:03,040 NOT BE A PLACEBO CONTROL ARM. 3926 02:35:03,040 --> 02:35:05,080 3927 02:35:05,080 --> 02:35:09,040 >> I'LL ADD AT THE NIH 3928 02:35:09,040 --> 02:35:11,880 INTRAMURAL WE CAN DO PHASE 1 3929 02:35:11,880 --> 02:35:13,440 MORE RISKY FOR ACADEMIC MEDICAL 3930 02:35:13,440 --> 02:35:13,760 CENTERS. 3931 02:35:13,760 --> 02:35:15,840 IT'S A VERY GOOD LEARNING 3932 02:35:15,840 --> 02:35:16,560 PROCESS. 3933 02:35:16,560 --> 02:35:18,880 WE CAN DO ADAPTIVE TRIAL DESIGN, 3934 02:35:18,880 --> 02:35:20,840 SMALL NUMBER OF PATIENTS, RARE 3935 02:35:20,840 --> 02:35:22,280 DISEASES, FROM THAT DATA 3936 02:35:22,280 --> 02:35:25,680 MAXIMIZE INFORMATION TO GO 3937 02:35:25,680 --> 02:35:28,520 FORWARD TO PHASE 1/2 TRIALS, 3938 02:35:28,520 --> 02:35:30,040 IT'S KEY ALLIANCE BETWEEN THE 3939 02:35:30,040 --> 02:35:33,320 PATIENT AND FAMILY ORGANIZATIONS 3940 02:35:33,320 --> 02:35:34,200 AND RESEARCHERS TO REALLY 3941 02:35:34,200 --> 02:35:35,480 OPTIMIZE FROM THE PERSPECTIVE OF 3942 02:35:35,480 --> 02:35:37,920 RARE DISEASE SO WE'RE NOT 3943 02:35:37,920 --> 02:35:39,240 DEALING WITH ADULT POPULATION OR 3944 02:35:39,240 --> 02:35:40,440 LARGE POPULATION. 3945 02:35:40,440 --> 02:35:42,640 I'M BEEN ON ADVISORY COMMITTEE 3946 02:35:42,640 --> 02:35:44,320 MEETINGS WHERE COLLEAGUES ARE 3947 02:35:44,320 --> 02:35:45,360 EXPERTS IN ALZHEIMER'S, VERY 3948 02:35:45,360 --> 02:35:48,360 IMPORTANT BUT A VERY DIFFERENT 3949 02:35:48,360 --> 02:35:54,360 PERSPECTIVE THAN FOR EXAMPLE 3950 02:35:54,360 --> 02:35:55,200 DUCHENNE MUSCULAR DYSTROPHY, 3951 02:35:55,200 --> 02:35:56,960 UNIFORMLY FATAL, TO TUNE IN THE 3952 02:35:56,960 --> 02:35:58,600 RADIO DIAL TO THAT PATIENT AND 3953 02:35:58,600 --> 02:36:00,480 FAMILY, UNDERSTAND WHAT THAT 3954 02:36:00,480 --> 02:36:02,840 LIFESPAN IS, WHAT LIFE IS LIKE, 3955 02:36:02,840 --> 02:36:03,640 IS ABSOLUTELY KEY. 3956 02:36:03,640 --> 02:36:08,800 THE OTHER PART OF THAT TOO WHEN 3957 02:36:08,800 --> 02:36:13,720 YOU ARE -- HOW DO I PUT THIS? 3958 02:36:13,720 --> 02:36:17,680 WHEN YOU ARE TALKING ABOUT A 3959 02:36:17,680 --> 02:36:21,960 DISEASE LIKE MLD, OR A DISEASE 3960 02:36:21,960 --> 02:36:25,160 THAT IS JUST SO -- WELL, NIH IS 3961 02:36:25,160 --> 02:36:27,840 A CRITICAL TOOL. 3962 02:36:27,840 --> 02:36:29,600 I WAS JOKING THAT YOU'RE 3963 02:36:29,600 --> 02:36:31,800 TERRIBLE AT SELF-PROMOTION. 3964 02:36:31,800 --> 02:36:34,760 THE FIRST GENE THERAPY IN THE 3965 02:36:34,760 --> 02:36:38,040 WORLD HAPPENED HERE WITH ADA 3966 02:36:38,040 --> 02:36:44,280 SCID, AND FRENCH ANDERSON AND 3967 02:36:44,280 --> 02:36:45,840 ACHANTE D'SILVA, I HAVE A 3968 02:36:45,840 --> 02:36:47,040 PHOTOGRAPH OF THE 40-YEAR-OLD, 3969 02:36:47,040 --> 02:36:48,680 THE FIRST CHILD EVER TREATED 3970 02:36:48,680 --> 02:36:51,320 WITH GENE THERAPY HERE AT THE 3971 02:36:51,320 --> 02:36:53,760 NIH BECAUSE DR. ANDERSON AND HIS 3972 02:36:53,760 --> 02:36:55,240 TEAM BELIEVED THEY HAD A 3973 02:36:55,240 --> 02:36:57,960 SCIENTIFIC CONCEPT THAT COULD 3974 02:36:57,960 --> 02:36:58,160 WORK. 3975 02:36:58,160 --> 02:37:01,720 THEY WENT THROUGH 20 HOURS OF 3976 02:37:01,720 --> 02:37:03,240 PUBLIC HEARINGS TO GET APPROVAL, 3977 02:37:03,240 --> 02:37:04,600 WENT THROUGH THE FDA. 3978 02:37:04,600 --> 02:37:08,960 THE NIH DID ALL THIS WORK AND 3979 02:37:08,960 --> 02:37:11,160 ASHANTE WAS NOT A CANDIDATE FOR 3980 02:37:11,160 --> 02:37:16,080 BONE MARROW TRANSPLANT, THE 3981 02:37:16,080 --> 02:37:17,680 FAMILY HAD THE TOLERABLE RISK 3982 02:37:17,680 --> 02:37:19,120 THRESHOLD, AND THE NIH MADE THAT 3983 02:37:19,120 --> 02:37:19,400 POSSIBLE. 3984 02:37:19,400 --> 02:37:21,200 I THINK THE NIH IF WE GO BACK 3985 02:37:21,200 --> 02:37:29,200 HAS BEEN DOING THIS REALLY WELL 3986 02:37:29,200 --> 02:37:30,840 FOR ALMOST 40 YEARS. 3987 02:37:30,840 --> 02:37:34,560 WE NEED TO GIVE THE NIH 3988 02:37:34,560 --> 02:37:37,120 RESOURCES TOOLS, CONGRESS, 3989 02:37:37,120 --> 02:37:38,600 PRESIDENTS, FUND. 3990 02:37:38,600 --> 02:37:40,640 WE CAN'T ASK INDUSTRY AND CAN'T 3991 02:37:40,640 --> 02:37:43,040 ASK FAMILIES TO DO THIS, AS MUCH 3992 02:37:43,040 --> 02:37:44,200 AS AMAZING FAMILIES DO GREAT 3993 02:37:44,200 --> 02:37:44,440 WORK. 3994 02:37:44,440 --> 02:37:49,000 THERE ARE LIMITS TO WHAT WE CAN 3995 02:37:49,000 --> 02:37:52,400 DO. 3996 02:37:52,400 --> 02:37:53,360 3997 02:37:53,360 --> 02:37:54,680 >> WE HAVE A QUESTION. 3998 02:37:54,680 --> 02:38:05,200 THIS WILL BE OUR LAST QUESTION. 3999 02:38:07,720 --> 02:38:08,720 >> SHARON ROSSI, BRIGHT FOCUS. 4000 02:38:08,720 --> 02:38:11,000 I WASN'T SURE IF THIS WAS A 4001 02:38:11,000 --> 02:38:12,520 QUESTION FOR BEFORE OR NOW. 4002 02:38:12,520 --> 02:38:16,440 JUST INTERESTED IN YOUR THOUGHTS 4003 02:38:16,440 --> 02:38:17,440 ESPECIALLY BECAUSE ALZHEIMER'S 4004 02:38:17,440 --> 02:38:21,480 DISEASE SPACE, ON THE FDA MAYBE 4005 02:38:21,480 --> 02:38:24,400 ROLLING BACK NEED TO USE ANIMALS 4006 02:38:24,400 --> 02:38:25,680 FOR CLINICAL TRIALS, HOW THAT 4007 02:38:25,680 --> 02:38:26,960 MIGHT BE DIFFERENT FROM WHAT I 4008 02:38:26,960 --> 02:38:28,400 HEAR IN THE ALZHEIMER'S SPACE TO 4009 02:38:28,400 --> 02:38:29,400 RARE DISEASES THAT WE'RE TALKING 4010 02:38:29,400 --> 02:38:32,160 ABOUT TODAY. 4011 02:38:32,160 --> 02:38:40,400 4012 02:38:40,400 --> 02:38:41,120 4013 02:38:41,120 --> 02:38:44,400 >> FROM MY UNDERSTANDING, IT'S 4014 02:38:44,400 --> 02:38:46,000 VERY DISEASE-AREA SPECIFIC. 4015 02:38:46,000 --> 02:38:49,680 AND WHAT OTHER MODELS WOULD BE 4016 02:38:49,680 --> 02:38:51,240 AVAILABLE TO COMPENSATE. 4017 02:38:51,240 --> 02:38:55,400 SO YOU CAN USE ANIMAL MODELS TO 4018 02:38:55,400 --> 02:38:57,480 TEST FOR EFFICACY, DOES YOUR 4019 02:38:57,480 --> 02:39:00,560 THERAPY WORK, IN THE ANIMAL 4020 02:39:00,560 --> 02:39:01,840 MODEL, DOES IT ALLEVIATE 4021 02:39:01,840 --> 02:39:02,760 SYMPTOMS BUT ALSO ANIMAL MODELS 4022 02:39:02,760 --> 02:39:09,960 COULD BE USED FOR TESTING, JUST 4023 02:39:09,960 --> 02:39:11,400 REGULAR ANIMALS. 4024 02:39:11,400 --> 02:39:13,400 SO THERE ARE DISCUSSIONS ABOUT 4025 02:39:13,400 --> 02:39:14,360 WHETHER OR NOT NON-HUMAN 4026 02:39:14,360 --> 02:39:16,600 PRIMATES FOR EXAMPLE MAY BE 4027 02:39:16,600 --> 02:39:18,000 NECESSARY FOR TOX STUDIES AND 4028 02:39:18,000 --> 02:39:20,320 CERTAIN TYPES OF GENE THERAPY 4029 02:39:20,320 --> 02:39:23,040 BUT I THINK IT'S VERY 4030 02:39:23,040 --> 02:39:26,080 DISEASE-AREA SPECIFIC. 4031 02:39:26,080 --> 02:39:30,680 AND WHAT -- YOU KNOW, INDUCE THE 4032 02:39:30,680 --> 02:39:32,440 PLURIPOTENT STEM CELLS ARE 4033 02:39:32,440 --> 02:39:35,360 AVAILABLE OR ORGANOID MODELS, I 4034 02:39:35,360 --> 02:39:37,440 THINK IT'S REALLY HARD TO SAY, 4035 02:39:37,440 --> 02:39:41,520 AS IT'S A SPECIFIC DISEASE AREA 4036 02:39:41,520 --> 02:39:45,680 >> I THINK FROM WHAT WE DEAL 4037 02:39:45,680 --> 02:39:46,440 WITH, PEDIATRIC DISEASES, IT'S 4038 02:39:46,440 --> 02:39:49,080 ESSENTIAL WE HAVE THE JUVENILE 4039 02:39:49,080 --> 02:39:51,920 TOXICOLOGY DATA SO FOR US IT'S 4040 02:39:51,920 --> 02:39:53,640 NECESSARY, AND NON-HUMAN PRIMATE 4041 02:39:53,640 --> 02:39:55,520 DATA AS WELL. 4042 02:39:55,520 --> 02:39:58,080 BUT IT'S A GREAT QUESTION. 4043 02:39:58,080 --> 02:40:03,520 WE SAID WE WOULD BE PROVOCATIVE, 4044 02:40:03,520 --> 02:40:03,880 DIDN'T WE? 4045 02:40:03,880 --> 02:40:08,080 >> I WANT TO THANK EACH OF THE 4046 02:40:08,080 --> 02:40:09,320 SPEAKERS TODAY. 4047 02:40:09,320 --> 02:40:11,800 I THINK THEY DID GREAT JOB WITH 4048 02:40:11,800 --> 02:40:15,120 TOUGH QUESTIONS RELATED TO THE 4049 02:40:15,120 --> 02:40:19,760 ETHICS AND CHALLENGES AND GENE 4050 02:40:19,760 --> 02:40:20,200 THERAPY. 4051 02:40:20,200 --> 02:40:25,400 [APPLAUSE] 4052 02:40:25,400 --> 02:40:28,880 4053 02:40:28,880 --> 02:40:31,640 I'M HANDING OFF TO OUR -- 4054 02:40:31,640 --> 02:40:36,800 REBECCA IS LOOKING AT ME. 4055 02:40:36,800 --> 02:40:38,960 THE NEXT MODERATOR CAN JOIN. 4056 02:40:38,960 --> 02:40:40,520 >> AND PANELISTS AS WELL. 4057 02:40:40,520 --> 02:41:00,400 >> THANKS SO MUCH. 4058 02:41:00,400 --> 02:41:01,520 >>I'M PENNY DAKS, FRONTOTEMPORAL 4059 02:41:01,520 --> 02:41:03,080 ORGANIZATION, WE'RE GOING TO 4060 02:41:03,080 --> 02:41:09,480 TALK ABOUT DIFFERENT STRATEGIES 4061 02:41:09,480 --> 02:41:12,400 THAT NON-PROFITS TAKE TO FUND 4062 02:41:12,400 --> 02:41:12,800 RESEARCH. 4063 02:41:12,800 --> 02:41:15,280 SO DIFFERENT THAN THE MORNING'S 4064 02:41:15,280 --> 02:41:17,920 DISCUSSION BUT A CENTRAL PART TO 4065 02:41:17,920 --> 02:41:21,160 WHAT NON-PROFITS SEEK TO DO. 4066 02:41:21,160 --> 02:41:24,360 AS NON-PROFITS WE HAVE TO 4067 02:41:24,360 --> 02:41:26,960 REMEMBER WE'RE A SMALL PIECE OF 4068 02:41:26,960 --> 02:41:30,680 A LARGE ECOSYSTEM FOR ADVANCING 4069 02:41:30,680 --> 02:41:33,320 TREATMENTS IN DIAGNOSTICS FOR 4070 02:41:33,320 --> 02:41:33,720 OUR CONDITIONS. 4071 02:41:33,720 --> 02:41:36,160 AND THE INTENTION OF TODAY'S 4072 02:41:36,160 --> 02:41:38,080 SESSION IS TO REVIEW BOTH 4073 02:41:38,080 --> 02:41:39,240 RESOURCES AND DIFFERENT 4074 02:41:39,240 --> 02:41:41,720 STRATEGIES THAT NON-PROFITS TAKE 4075 02:41:41,720 --> 02:41:50,080 TO FIND WAYS TO MOVE THE NEEDLE. 4076 02:41:50,080 --> 02:41:52,800 I SAW ANOTHER PERSON PRESENT ON 4077 02:41:52,800 --> 02:41:55,640 THE SIZE OF NON-PROFIT FUNDING 4078 02:41:55,640 --> 02:41:57,720 COMPARED TO NINDS. 4079 02:41:57,720 --> 02:41:59,240 IT IS QUITE BREATHTAKING BUT 4080 02:41:59,240 --> 02:42:01,200 MAKES IT SO PIVOTAL WE THINK 4081 02:42:01,200 --> 02:42:03,720 CAREFULLY HOW WE DESIGN OUR 4082 02:42:03,720 --> 02:42:05,400 FUNDING PORTFOLIOS TO FIND THE 4083 02:42:05,400 --> 02:42:07,680 GAPS THAT ARE NON-EASILY DONE BY 4084 02:42:07,680 --> 02:42:09,760 THE NIH AND LEVERAGE THE 4085 02:42:09,760 --> 02:42:12,920 RESOURCES OF LARGER FUNDERS LIKE 4086 02:42:12,920 --> 02:42:13,680 THE NIH. 4087 02:42:13,680 --> 02:42:16,080 SO I'M THRILLED WE HAVE A GREAT 4088 02:42:16,080 --> 02:42:17,960 SET OF PANELISTS HERE. 4089 02:42:17,960 --> 02:42:25,600 WE'LL START WITH CHRIS TORBORG 4090 02:42:25,600 --> 02:42:27,480 TO DESCRIBE HOW NIH RESOURCES 4091 02:42:27,480 --> 02:42:30,400 LIKE THE FEDERAL REPORTER CAN 4092 02:42:30,400 --> 02:42:31,640 HELP UNDERSTAND THE FUNDING 4093 02:42:31,640 --> 02:42:36,920 LANDSCAPE FOR ANY GIVEN 4094 02:42:36,920 --> 02:42:38,120 CONDITION. 4095 02:42:38,120 --> 02:42:42,040 AND THEN WE'LL HEAR ABOUT NOT 4096 02:42:42,040 --> 02:42:45,240 THE WHOLE PORTFOLIO, IT'S REALLY 4097 02:42:45,240 --> 02:42:46,040 SPECIFIC TO ONE OR TWO 4098 02:42:46,040 --> 02:42:48,080 STRATEGIES THAT THEY ARE TAKING 4099 02:42:48,080 --> 02:42:51,280 THAT IS A DIFFERENT APPROACH TO 4100 02:42:51,280 --> 02:42:55,000 TRY TO FUND RESEARCH. 4101 02:42:55,000 --> 02:42:58,280 WITH THAT I'LL TEE UP THE NEXT 4102 02:42:58,280 --> 02:42:58,520 SPEAKER. 4103 02:42:58,520 --> 02:43:01,760 SO DR. TORBORG. 4104 02:43:01,760 --> 02:43:04,240 >> SO THANKS. 4105 02:43:04,240 --> 02:43:05,320 I'M CHRISTINE TORBORG, OFFICE OF 4106 02:43:05,320 --> 02:43:06,840 SCIENCE POLICY AND PLANNING. 4107 02:43:06,840 --> 02:43:10,000 WE DO A WIDE VARIETY OF POLICY 4108 02:43:10,000 --> 02:43:11,560 ISSUES ACROSS FOR NINDS. 4109 02:43:11,560 --> 02:43:17,680 ONE OF THE BIG ISSUES WE WORK ON 4110 02:43:17,680 --> 02:43:18,640 ARE ANALYSIS OF PORTFOLIOS TO 4111 02:43:18,640 --> 02:43:19,320 UNDERSTAND IT. 4112 02:43:19,320 --> 02:43:21,600 I'M GOING TO GIVE A BROAD REVIEW 4113 02:43:21,600 --> 02:43:24,240 OF WHAT SOME TOOLS ARE, AND 4114 02:43:24,240 --> 02:43:25,360 THERE'S A LOT. 4115 02:43:25,360 --> 02:43:30,600 FIVE MINUTES IS HARD TO COVER IT 4116 02:43:30,600 --> 02:43:30,840 ALL. 4117 02:43:30,840 --> 02:43:33,760 THE ONE STOP SHOP THAT EVERYBODY 4118 02:43:33,760 --> 02:43:44,280 SHOULD KNOW OF IS NIH REPORT, 4119 02:43:44,560 --> 02:43:44,880 REPORT.NIH.GOV. 4120 02:43:44,880 --> 02:43:50,520 I TOOK A SCREEN SHOT OF THE HOME 4121 02:43:50,520 --> 02:43:50,720 PAGE. 4122 02:43:50,720 --> 02:43:56,640 YOU GO ACROSS THE TOP, START 4123 02:43:56,640 --> 02:43:57,320 CONTROLLING SCROLLING. 4124 02:43:57,320 --> 02:43:58,720 THERE'S LINKS TO EVERYTHING. 4125 02:43:58,720 --> 02:44:01,960 YOU CAN FIND VARIOUS TYPES OF 4126 02:44:01,960 --> 02:44:03,960 STRATEGIC PLANS ACROSS ALL OF 4127 02:44:03,960 --> 02:44:04,880 NIH, DIFFERENT STRATEGIC PLANS 4128 02:44:04,880 --> 02:44:07,360 CAN BE LINKED UNDER THE REPORTS 4129 02:44:07,360 --> 02:44:07,800 TOOL. 4130 02:44:07,800 --> 02:44:08,440 THERE'S WORKFORCE. 4131 02:44:08,440 --> 02:44:09,680 THERE'S ALL SORTS OF REPORTS 4132 02:44:09,680 --> 02:44:13,600 FROM NIH OFFICE OF DIRECTOR ON 4133 02:44:13,600 --> 02:44:15,000 THE BIOMEDICAL RESEARCH 4134 02:44:15,000 --> 02:44:16,560 WORKFORCE, WHAT'S NEEDED. 4135 02:44:16,560 --> 02:44:19,640 THERE'S A HUGE PLETHORA OF TOOLS 4136 02:44:19,640 --> 02:44:20,760 THERE TO LOOK. 4137 02:44:20,760 --> 02:44:22,880 I THINK FOR THE PURPOSES OF 4138 02:44:22,880 --> 02:44:23,800 UNDERSTANDING PORTFOLIO THERE'S 4139 02:44:23,800 --> 02:44:28,520 A FEW TOOLS MOST USEFUL FOR YOU. 4140 02:44:28,520 --> 02:44:34,080 ONE IS THAT WE HAVE THE NIH DATA 4141 02:44:34,080 --> 02:44:35,920 BOOK. 4142 02:44:35,920 --> 02:44:37,480 PRE-PREPARED REPORTS OF THE 4143 02:44:37,480 --> 02:44:38,160 NIH -- FREQUENTLY ASKED 4144 02:44:38,160 --> 02:44:40,680 QUESTIONS FOR NIH DATA. 4145 02:44:40,680 --> 02:44:44,280 THESE ARE ACROSS NIH, SORT OF 4146 02:44:44,280 --> 02:44:46,920 LIKE HOW MANY TRAINING GRANTS IS 4147 02:44:46,920 --> 02:44:48,880 NIH FUNDING, ET CETERA. 4148 02:44:48,880 --> 02:44:51,280 YOU CAN LOOK FOR THOSE REPORTS. 4149 02:44:51,280 --> 02:44:54,880 CATEGORICAL SPENDING IS REALLY 4150 02:44:54,880 --> 02:44:57,840 ABOUT THOSE CATEGORIES OR 4151 02:44:57,840 --> 02:44:58,320 SCIENTIFIC AND DISEASE 4152 02:44:58,320 --> 02:45:01,560 CATEGORIES WE'RE REQUIRED TO 4153 02:45:01,560 --> 02:45:02,640 REPORT TO CONGRESS, PUBLICLY 4154 02:45:02,640 --> 02:45:03,200 AVAILABLE DATA. 4155 02:45:03,200 --> 02:45:05,400 TODAY I WANT TO TALK MORE ABOUT 4156 02:45:05,400 --> 02:45:11,080 THE NIH REPORTER DATABASE, AN 4157 02:45:11,080 --> 02:45:16,480 INCREDIBLY POWERFUL TOOL FOR 4158 02:45:16,480 --> 02:45:17,720 IDENTIFYING GRANTS OF RELEVANCE. 4159 02:45:17,720 --> 02:45:19,600 HOPEFULLY IT'S EASY TO FIND. 4160 02:45:19,600 --> 02:45:21,800 IT ALLOWS YOU TO SEARCH ON A 4161 02:45:21,800 --> 02:45:23,120 WIDE VARIETY OF SEARCH FIELDS 4162 02:45:23,120 --> 02:45:27,760 SUCH AS P.I., RESEARCH 4163 02:45:27,760 --> 02:45:29,600 INSTITUTIONS, KEY WORDS, TEXT 4164 02:45:29,600 --> 02:45:35,000 BASE SEARCHES, ALL SORTS OF 4165 02:45:35,000 --> 02:45:35,200 THINGS. 4166 02:45:35,200 --> 02:45:37,240 IT HAS DATA BACK TO 1985. 4167 02:45:37,240 --> 02:45:38,880 YOU CAN LOOK AT CHANGES OVER 4168 02:45:38,880 --> 02:45:39,360 TIME. 4169 02:45:39,360 --> 02:45:43,280 THERE'S A HUGE NUMBER OF 4170 02:45:43,280 --> 02:45:43,640 ANALYSES. 4171 02:45:43,640 --> 02:45:45,040 SOME TOOLS IMPLEMENTED MORE 4172 02:45:45,040 --> 02:45:47,440 RECENTLY THAT ARE INTERESTING IS 4173 02:45:47,440 --> 02:45:48,360 THE MATCHMAKER FUNCTION. 4174 02:45:48,360 --> 02:45:51,720 IF YOU GO OVER HERE TO THE 4175 02:45:51,720 --> 02:45:53,560 MATCHMAKER AND GET STARTED, THAT 4176 02:45:53,560 --> 02:45:58,240 TOOL ALLOWS YOU TO CUT AND PASTE 4177 02:45:58,240 --> 02:46:00,000 ABSTRACT, IT WILL THEN FIND NIH 4178 02:46:00,000 --> 02:46:01,480 GRANTS THAT HAVE SIMILAR TOPICS 4179 02:46:01,480 --> 02:46:02,200 TO THAT ABSTRACT. 4180 02:46:02,200 --> 02:46:05,920 YOU CAN GET A LIST OF GRANTS. 4181 02:46:05,920 --> 02:46:07,000 FOR EXAMPLE, IF YOU'RE 4182 02:46:07,000 --> 02:46:08,640 PERHAPS -- IF YOU HAVE A 4183 02:46:08,640 --> 02:46:09,840 RESEARCH PROGRAM, SUPPOSED TO 4184 02:46:09,840 --> 02:46:12,680 GIVE OUT GRANTS, YOU MAY WANT TO 4185 02:46:12,680 --> 02:46:14,400 PUT IN THE ABSTRACT THIS TOOL TO 4186 02:46:14,400 --> 02:46:17,280 SEE WHAT ELSE NIH IS THERE BUT 4187 02:46:17,280 --> 02:46:18,080 TO IDENTIFY POTENTIAL 4188 02:46:18,080 --> 02:46:19,040 INVESTIGATORS WHO ARE DOING 4189 02:46:19,040 --> 02:46:20,680 SIMILAR RESEARCH WHO MIGHT BE 4190 02:46:20,680 --> 02:46:23,120 GOOD TO REVIEW THAT GRANT IF YOU 4191 02:46:23,120 --> 02:46:23,960 WANT TO. 4192 02:46:23,960 --> 02:46:28,560 WE USE THAT AT NIH. 4193 02:46:28,560 --> 02:46:29,840 THERE'S ALSO A PUBLICATION 4194 02:46:29,840 --> 02:46:33,240 SEARCH TO PUT IN THE PUBLIC MED 4195 02:46:33,240 --> 02:46:36,640 I.D., A PMID, ON PubMed, FOR 4196 02:46:36,640 --> 02:46:38,600 PUBLICATION, AND IT WILL SPIT 4197 02:46:38,600 --> 02:46:43,320 BACK, GIVE YOU BACK A LIST OF 4198 02:46:43,320 --> 02:46:45,280 THE GRANTS ASSOCIATED WITH THAT 4199 02:46:45,280 --> 02:46:45,640 PUBLICATION. 4200 02:46:45,640 --> 02:46:49,800 WHICH COULD ALSO BE HELPFUL. 4201 02:46:49,800 --> 02:46:51,240 THIS DATABASE INCLUDES DATA NOT 4202 02:46:51,240 --> 02:46:55,680 JUST FROM NIH BUT SOME DATA FROM 4203 02:46:55,680 --> 02:47:00,080 FDA, CDC, VA, AGENCY FOR 4204 02:47:00,080 --> 02:47:03,360 CHILDREN AND FAMILIES, AHRQ, AND 4205 02:47:03,360 --> 02:47:06,840 HRSA, I'M BLANKING ON WHAT IT 4206 02:47:06,840 --> 02:47:08,480 STANDS FOR. 4207 02:47:08,480 --> 02:47:09,800 IT'S ANOTHER FEDERAL AGENCY THAT 4208 02:47:09,800 --> 02:47:11,640 FUNDS RESEARCH. 4209 02:47:11,640 --> 02:47:14,920 THE DATA IS OFTEN NOT AS 4210 02:47:14,920 --> 02:47:15,600 COMPLETE AS NIH. 4211 02:47:15,600 --> 02:47:20,440 IF A GRANT IS NOT THERE IT 4212 02:47:20,440 --> 02:47:22,160 DOESN'T MEAN -- IF IT IS THERE 4213 02:47:22,160 --> 02:47:25,760 YOU CAN GET MORE INFORMATION. 4214 02:47:25,760 --> 02:47:28,400 NIH REPORTER IS CONSTANTLY 4215 02:47:28,400 --> 02:47:30,080 UPDATED, GRANTS APPEAR WITHIN 4216 02:47:30,080 --> 02:47:33,000 REPORTER WITHIN 7 TO 10 DAYS OF 4217 02:47:33,000 --> 02:47:35,200 BUDGET START DATE. 4218 02:47:35,200 --> 02:47:38,280 IT'S UPDATED WEEKLY. 4219 02:47:38,280 --> 02:47:39,800 USUALLY MONDAYS, THE BIG UPLOAD 4220 02:47:39,800 --> 02:47:43,200 AT NIH. 4221 02:47:43,200 --> 02:47:45,400 IF YOU DO DO A SEARCH, WHAT DO 4222 02:47:45,400 --> 02:47:46,280 YOU GET OUT? 4223 02:47:46,280 --> 02:47:49,440 WHAT I DID HERE, A SEARCH ON 4224 02:47:49,440 --> 02:47:50,560 ACTIVE GRANTS THAT HAVE 4225 02:47:50,560 --> 02:47:52,640 PARKINSON'S DISEASE IN THE 4226 02:47:52,640 --> 02:47:52,920 TITLE. 4227 02:47:52,920 --> 02:48:01,120 AND THE RESULT IS I GOT 182 4228 02:48:01,120 --> 02:48:01,400 PROJECTS. 4229 02:48:01,400 --> 02:48:03,560 YOU GET A LIST OF THE PROJECTS. 4230 02:48:03,560 --> 02:48:05,640 YOU CAN DO ADDITIONAL FILTERING 4231 02:48:05,640 --> 02:48:08,360 FOR THE FISCAL YEARS OR 4232 02:48:08,360 --> 02:48:10,760 ORGANIZATIONS, ET CETERA. 4233 02:48:10,760 --> 02:48:11,400 YOU CAN CREATE AUTOMATIC CHARTS 4234 02:48:11,400 --> 02:48:12,320 OF THE PROJECTS. 4235 02:48:12,320 --> 02:48:14,480 FOR EXAMPLE, YOU MIGHT WANT A 4236 02:48:14,480 --> 02:48:17,640 CHART TO SAY OF THE 182 GRANTS 4237 02:48:17,640 --> 02:48:19,320 WITH PARKINSON'S DISEASE IN THE 4238 02:48:19,320 --> 02:48:24,440 TITLE WHAT FRACTION WERE FUNDED 4239 02:48:24,440 --> 02:48:26,240 BY NINDS VERSUS NATIONAL 4240 02:48:26,240 --> 02:48:27,080 INSTITUTE ON AGING, THERE'S 4241 02:48:27,080 --> 02:48:29,720 AUTOMATIC CHARTS THAT CAN BE 4242 02:48:29,720 --> 02:48:29,920 MADE. 4243 02:48:29,920 --> 02:48:32,760 YOU CAN CREATE A MAP OF THE 4244 02:48:32,760 --> 02:48:35,200 UNITED STATES WITH WHERE ALL THE 4245 02:48:35,200 --> 02:48:36,520 GRANTS ACROSS THE COUNTRY. 4246 02:48:36,520 --> 02:48:43,600 THERE'S ALSO SOME TOOLS FOR 4247 02:48:43,600 --> 02:48:44,120 PUBLICATIONS, PATENTS, 4248 02:48:44,120 --> 02:48:47,560 PUBLICATIONS ASSOCIATED WITH THE 4249 02:48:47,560 --> 02:48:47,800 GRANTS. 4250 02:48:47,800 --> 02:48:49,000 YOU CAN CLICK ON INDIVIDUAL 4251 02:48:49,000 --> 02:48:56,560 GRANTS AND GET MORE INFORMATION 4252 02:48:56,560 --> 02:48:57,480 ABOUT INDIVIDUAL GRANTS. 4253 02:48:57,480 --> 02:49:02,800 YOU CAN EXPORT ALL OF THIS DATA 4254 02:49:02,800 --> 02:49:05,640 INTO FILES USING THE EXHORT 4255 02:49:05,640 --> 02:49:06,680 TOOL, HUGE NUMBERS OF DATA 4256 02:49:06,680 --> 02:49:12,320 FIELDS IF YOU WANT TO DO OTHER 4257 02:49:12,320 --> 02:49:14,560 FURTHER ANALYSES IN EXCEL. 4258 02:49:14,560 --> 02:49:15,680 WHEN YOU'RE LOOKING AT 4259 02:49:15,680 --> 02:49:17,440 INDIVIDUAL GRANTS THE TYPES OF 4260 02:49:17,440 --> 02:49:18,960 INFORMATION FOR THOSE GRANTS ARE 4261 02:49:18,960 --> 02:49:21,280 ALL ABOUT THE PROJECT 4262 02:49:21,280 --> 02:49:23,920 DESCRIPTIONS, THE FULL ABSTRACT, 4263 02:49:23,920 --> 02:49:24,920 PUBLIC HEALTH RELEVANCE 4264 02:49:24,920 --> 02:49:26,200 STATEMENT, PROJECT TERMS, 4265 02:49:26,200 --> 02:49:28,200 DETAILS ABOUT THE FUNDING, P.I., 4266 02:49:28,200 --> 02:49:30,400 ORGANIZATION, WHO THE PROGRAM 4267 02:49:30,400 --> 02:49:31,600 OFFICIAL AT NIH IS. 4268 02:49:31,600 --> 02:49:32,920 FOR EXAMPLE IF YOU WANT TO KNOW 4269 02:49:32,920 --> 02:49:37,120 WHO IS LOOKING AT LET'S SAY 4270 02:49:37,120 --> 02:49:39,800 GENETICS AND PARKINSON'S 4271 02:49:39,800 --> 02:49:41,360 DISEASE, WHO DO I CONTACT? 4272 02:49:41,360 --> 02:49:46,520 LOOK AT GRANTS AND SEE WHO IS 4273 02:49:46,520 --> 02:49:48,360 THE MOST FREQUENTLY CITED 4274 02:49:48,360 --> 02:49:49,920 PROGRAM OFFICIAL, THE PERSON YOU 4275 02:49:49,920 --> 02:49:51,960 PROBABLY WANT TO CONTACT. 4276 02:49:51,960 --> 02:49:53,520 OR WHAT STUDY SECTIONS ARE THEY 4277 02:49:53,520 --> 02:49:54,760 BEING REVIEWED IN. 4278 02:49:54,760 --> 02:49:57,000 YOU CAN GET A LOT OF INFORMATION 4279 02:49:57,000 --> 02:50:03,040 ABOUT RESULTS OF THOSE STUDIES. 4280 02:50:03,040 --> 02:50:06,040 FOR EXAMPLE, IT HAS LINKS TO 4281 02:50:06,040 --> 02:50:07,760 PubMed WITH ALL OF THE 4282 02:50:07,760 --> 02:50:09,960 GRANTS THAT ARE THE PUBLICATIONS 4283 02:50:09,960 --> 02:50:11,600 THAT CITED THAT GRANT. 4284 02:50:11,600 --> 02:50:13,240 IF THERE'S A PATENT YOU CAN GET 4285 02:50:13,240 --> 02:50:16,400 A LINK TO PATENT OFFICE FOR THAT 4286 02:50:16,400 --> 02:50:19,560 GRANT, FOR THAT PATENT. 4287 02:50:19,560 --> 02:50:20,760 THERE ARE OTHER OUTCOMES. 4288 02:50:20,760 --> 02:50:22,160 I'VE NEVER SEEN THIS DATA FIELD 4289 02:50:22,160 --> 02:50:24,040 FILLED SO I DON'T KNOW WHAT GOES 4290 02:50:24,040 --> 02:50:25,280 IN THERE. 4291 02:50:25,280 --> 02:50:26,880 IT LINKS IF THERE ARE CLINICAL 4292 02:50:26,880 --> 02:50:28,320 TRIALS OR CLINICAL STUDIES 4293 02:50:28,320 --> 02:50:29,400 ASSOCIATED WITH THAT GRANT, 4294 02:50:29,400 --> 02:50:31,280 LINKS TO clinicaltrials.gov TO 4295 02:50:31,280 --> 02:50:33,440 GIVE MORE INFORMATION ABOUT THE 4296 02:50:33,440 --> 02:50:34,560 CLINICAL TRIALS. 4297 02:50:34,560 --> 02:50:36,200 IT CAN LINK TO PRESS RELEASES 4298 02:50:36,200 --> 02:50:37,520 WHEN THEY ARE OUT THERE. 4299 02:50:37,520 --> 02:50:40,680 CERTAINLY THE NIH PUT OUT A 4300 02:50:40,680 --> 02:50:41,880 PRESS RELEASE FROM STUDIES BASED 4301 02:50:41,880 --> 02:50:43,080 ON THAT GRANT. 4302 02:50:43,080 --> 02:50:45,080 YOU CAN GET HISTORY OF THE 4303 02:50:45,080 --> 02:50:46,280 GRANT, WHAT'S HAPPENED IN 4304 02:50:46,280 --> 02:50:47,120 PREVIOUS YEARS. 4305 02:50:47,120 --> 02:50:50,520 IT ALSO HAS A SIMILAR PROJECT 4306 02:50:50,520 --> 02:50:52,600 WHICH DOES THAT MATCHING TOOL. 4307 02:50:52,600 --> 02:50:54,120 THIS IS AN OVERVIEW OF THE DATA 4308 02:50:54,120 --> 02:50:57,200 THAT'S THERE. 4309 02:50:57,200 --> 02:50:58,760 YOU REALLY HAVE TO EXPLORE TO 4310 02:50:58,760 --> 02:51:00,360 SEE WHAT WORKS FOR YOU. 4311 02:51:00,360 --> 02:51:02,560 I DID WANT TO SAY A FEW 4312 02:51:02,560 --> 02:51:04,080 RESOURCES TO HELP YOU UNDERSTAND 4313 02:51:04,080 --> 02:51:07,400 IT BETTER, IT'S A LOT, SO THERE 4314 02:51:07,400 --> 02:51:08,600 ARE FREQUENTLY ASKED QUESTIONS 4315 02:51:08,600 --> 02:51:10,760 PAGES THROUGHOUT THE SITE YOU 4316 02:51:10,760 --> 02:51:13,040 MAY WANT TO INVESTIGATE AND 4317 02:51:13,040 --> 02:51:13,280 EXPLORE. 4318 02:51:13,280 --> 02:51:16,040 THERE'S ALSO, IF YOU GO TO 4319 02:51:16,040 --> 02:51:20,840 EXTRAMURAL NEXUS, THE LINK HERE, 4320 02:51:20,840 --> 02:51:23,320 IT'S ALL ABOUT NIH FUNDING. 4321 02:51:23,320 --> 02:51:25,040 IT GIVES YOU A LOT OF TIPS HOW 4322 02:51:25,040 --> 02:51:29,240 TO USE REPORTER IF YOU SEARCH, 4323 02:51:29,240 --> 02:51:31,120 THE HISTORY OF THE BLOGS, HOW TO 4324 02:51:31,120 --> 02:51:31,640 USE THAT. 4325 02:51:31,640 --> 02:51:32,760 THERE'S OTHER THINGS YOU MIGHT 4326 02:51:32,760 --> 02:51:36,160 WANT TO EXPLORE AS WELL. 4327 02:51:36,160 --> 02:51:38,680 FINALLY THE NIH REPORT SITE HAS 4328 02:51:38,680 --> 02:51:41,320 A CONTACT E-MAIL THAT YOU CAN 4329 02:51:41,320 --> 02:51:44,280 CONTACT IF YOU NEED ADDITIONAL 4330 02:51:44,280 --> 02:51:44,720 HELP. 4331 02:51:44,720 --> 02:51:46,240 THAT'S ALL I WANTED TO SAY, TO 4332 02:51:46,240 --> 02:51:47,880 MAKE SURE YOU KNOW IT'S THERE. 4333 02:51:47,880 --> 02:51:49,240 THERE'S A LOT THERE AND YOU 4334 02:51:49,240 --> 02:51:51,080 SHOULD EXPLORE IT IF YOU HAVE A 4335 02:51:51,080 --> 02:51:53,360 CHANCE. 4336 02:51:53,360 --> 02:51:57,520 4337 02:51:57,520 --> 02:52:04,160 >> I WANT TO ASK ONE COMMENT. 4338 02:52:04,160 --> 02:52:06,280 I OFTEN SEE PEOPLE PARTICULARLY 4339 02:52:06,280 --> 02:52:08,200 YOU'RE LIVING WITH A DISEASE, GO 4340 02:52:08,200 --> 02:52:10,160 TO NIH REPORTER, YOU DON'T SEE 4341 02:52:10,160 --> 02:52:11,520 YOUR DISEASE FUNDED AT THE LEVEL 4342 02:52:11,520 --> 02:52:12,600 OF OTHER DISEASES. 4343 02:52:12,600 --> 02:52:14,600 I WONDER IF YOU WANT TO REFLECT 4344 02:52:14,600 --> 02:52:18,280 FOR A MOMENT, IS THAT BECAUSE 4345 02:52:18,280 --> 02:52:21,160 IT'S THE NIH ON THIS NOT THAT, 4346 02:52:21,160 --> 02:52:22,000 THERE'S RESEARCHERS APPLYING FOR 4347 02:52:22,000 --> 02:52:24,640 GRANTS AND THOSE GRANTS ARE 4348 02:52:24,640 --> 02:52:25,960 REVIEWED FOR QUALITY AND 4349 02:52:25,960 --> 02:52:27,360 SOMETIMES THERE ARE WHOLE FIELDS 4350 02:52:27,360 --> 02:52:28,680 WITH TONS OF RESEARCHERS WORKING 4351 02:52:28,680 --> 02:52:31,720 IN THEM AND THE ROOT CAUSE IS 4352 02:52:31,720 --> 02:52:34,720 NOT NECESSARILY A TOP-DOWN 4353 02:52:34,720 --> 02:52:37,400 DECISION. 4354 02:52:37,400 --> 02:52:38,960 >> ABSOLUTELY WHAT WE THINK IS 4355 02:52:38,960 --> 02:52:42,920 USUALLY GOING ON IS THAT IT'S 4356 02:52:42,920 --> 02:52:44,440 REALLY THERE HASN'T BEEN A 4357 02:52:44,440 --> 02:52:45,640 GROUNDSWELL IN THE RESEARCH 4358 02:52:45,640 --> 02:52:47,200 FIELD OR ENOUGH ADVANCES TO 4359 02:52:47,200 --> 02:52:49,480 ATTRACT MORE PEOPLE. 4360 02:52:49,480 --> 02:52:50,840 SOMETIMES FINDING A GENE, 4361 02:52:50,840 --> 02:52:53,800 TALKING ABOUT GENE THERAPY, CAN 4362 02:52:53,800 --> 02:52:57,920 REALLY NOW SEED A RESEARCH AREA. 4363 02:52:57,920 --> 02:52:59,680 THE OTHER THING I WANT PEOPLE TO 4364 02:52:59,680 --> 02:53:01,440 THINK ABOUT IS TO NOT THINK 4365 02:53:01,440 --> 02:53:03,640 NARROWLY ABOUT HOW YOU DO YOUR 4366 02:53:03,640 --> 02:53:06,480 SEARCHES BUT TO THINK MORE 4367 02:53:06,480 --> 02:53:07,440 BROADLY. 4368 02:53:07,440 --> 02:53:09,720 SOMETIMES DEPENDING ON WHAT 4369 02:53:09,720 --> 02:53:14,000 THERMOMETERS YOU USE, WHAT DO 4370 02:53:14,000 --> 02:53:14,560 YOU CONSIDER RESEARCH. 4371 02:53:14,560 --> 02:53:16,200 THERE'S THINGS ON THE PERIPHERY 4372 02:53:16,200 --> 02:53:17,520 THAT ARE RELEVANT. 4373 02:53:17,520 --> 02:53:21,000 WE DO A LOT OF CODING AND 4374 02:53:21,000 --> 02:53:25,000 THINKING ABOUT -- WE HAVE TO DO 4375 02:53:25,000 --> 02:53:26,480 SOME REPORTING, HOW MUCH YOU 4376 02:53:26,480 --> 02:53:28,040 SPEND, IT'S NEVER A CLEAR LINE 4377 02:53:28,040 --> 02:53:29,240 WHERE YOU CUT IT OFF. 4378 02:53:29,240 --> 02:53:32,080 I HAVE PARKINSON'S DISEASE ON 4379 02:53:32,080 --> 02:53:33,920 THE MIND BECAUSE I DO A LOT WITH 4380 02:53:33,920 --> 02:53:36,560 THAT RIGHT NOW. 4381 02:53:36,560 --> 02:53:38,960 WE HAVE GRANTS TO STUDY, TO 4382 02:53:38,960 --> 02:53:39,960 IDENTIFY GENES, FOR EXAMPLE, IN 4383 02:53:39,960 --> 02:53:42,360 PARKINSON'S DISEASE BUT WE ALSO 4384 02:53:42,360 --> 02:53:46,560 HAVE GRANTS TO UNDERSTAND THE 4385 02:53:46,560 --> 02:53:48,960 BASAL GANGLIA CIRCUIT, PARTS OF 4386 02:53:48,960 --> 02:53:50,560 THE BRAIN INVOLVED, STUDYING IN 4387 02:53:50,560 --> 02:53:53,200 A NORMAL PERSON, IT'S STILL 4388 02:53:53,200 --> 02:53:53,960 RELEVANT TO PARKINSON'S BECAUSE 4389 02:53:53,960 --> 02:53:56,920 YOU HAVE TO UNDERSTAND HOW THE 4390 02:53:56,920 --> 02:53:59,200 BRAIN NORMALLY WORKS TO KNOW HOW 4391 02:53:59,200 --> 02:54:01,280 IT GOES WRONG. 4392 02:54:01,280 --> 02:54:04,040 DO YOU INCLUDE THAT PARKINSON'S 4393 02:54:04,040 --> 02:54:05,120 DISEASE RESEARCH OR BASIC 4394 02:54:05,120 --> 02:54:06,680 SCIENCE HOW THE BRAIN WORKS? 4395 02:54:06,680 --> 02:54:08,080 BOTH ARE REALLY IMPORTANT. 4396 02:54:08,080 --> 02:54:09,840 THAT'S THE OTHER PART I WANTED 4397 02:54:09,840 --> 02:54:11,680 TO SAY ABOUT THAT. 4398 02:54:11,680 --> 02:54:13,720 >> THANK YOU. 4399 02:54:13,720 --> 02:54:15,400 NEXT UP IS DEBRA NIEHOFF FROM 4400 02:54:15,400 --> 02:54:16,840 AFTD. 4401 02:54:16,840 --> 02:54:19,280 >> THANK YOU AND GOOD AFTERNOON. 4402 02:54:19,280 --> 02:54:20,960 I'M DEBRA NIEHOFF, THE DIRECTOR 4403 02:54:20,960 --> 02:54:25,560 OF RESEARCH AND GRANTS FOR THE 4404 02:54:25,560 --> 02:54:26,840 ASSOCIATION FOR FRONTOTEMPORAL 4405 02:54:26,840 --> 02:54:28,200 DEGENERATION, AFTD. 4406 02:54:28,200 --> 02:54:29,560 I'M GRATEFUL FOR THE OPPORTUNITY 4407 02:54:29,560 --> 02:54:32,440 TO TELL YOU A LITTLE BIT ABOUT 4408 02:54:32,440 --> 02:54:36,480 ONE OF OUR MOST EFFECTIVE 4409 02:54:36,480 --> 02:54:37,600 FUNDING STRATEGIES. 4410 02:54:37,600 --> 02:54:41,960 FOR OVER 15 YEARS AFTD HAS 4411 02:54:41,960 --> 02:54:44,200 LOOKED TO OTHER NON-PROFIT 4412 02:54:44,200 --> 02:54:46,120 FUNDING ORGANIZATIONS AS 4413 02:54:46,120 --> 02:54:47,120 PARTNERS. 4414 02:54:47,120 --> 02:54:48,520 BY WORKING TOGETHER 4415 02:54:48,520 --> 02:54:49,600 COLLABORATIVELY, WE'VE BEEN ABLE 4416 02:54:49,600 --> 02:54:51,280 TO ACCOMPLISH GOALS THAT WOULD 4417 02:54:51,280 --> 02:54:52,440 HAVE BEEN VERY CHALLENGING IF WE 4418 02:54:52,440 --> 02:54:57,160 HAD TRIED TO FUND THEM ON OUR 4419 02:54:57,160 --> 02:54:57,480 OWN. 4420 02:54:57,480 --> 02:55:01,840 WE'VE HEARD A LOT ALL DAY ABOUT 4421 02:55:01,840 --> 02:55:04,640 THE VALUE OF PARTNERSHIPS 4422 02:55:04,640 --> 02:55:07,320 BETWEEN RESEARCHERS AND PATIENT 4423 02:55:07,320 --> 02:55:09,360 ADVOCACY ORGANIZATIONS. 4424 02:55:09,360 --> 02:55:11,840 I'M GOING TO GIVE HERE A 4425 02:55:11,840 --> 02:55:13,480 FUNDER'S PERSPECTIVE ON VALUE 4426 02:55:13,480 --> 02:55:17,680 ADD FOR FUNDING PARTNERSHIPS 4427 02:55:17,680 --> 02:55:20,560 PARTNERSHIPS CAN ALLOW 4428 02:55:20,560 --> 02:55:21,920 YOU TO ADD ADDITIONAL RESOURCES 4429 02:55:21,920 --> 02:55:22,720 TO EXISTING PROGRAMS. 4430 02:55:22,720 --> 02:55:24,840 YOU MIGHT BE ABLE TO FUND 4431 02:55:24,840 --> 02:55:26,240 ADDITIONAL AWARD IN A FUNDING 4432 02:55:26,240 --> 02:55:29,200 CYCLE BECAUSE THAT PROJECT HAS 4433 02:55:29,200 --> 02:55:32,800 RELEVANCE TO BOTH ORGANIZATIONS. 4434 02:55:32,800 --> 02:55:34,120 SECONDLY, FUNDING PARTNERSHIPS 4435 02:55:34,120 --> 02:55:37,160 HAVE ALLOWS US TO INITIATE NEW 4436 02:55:37,160 --> 02:55:40,920 PROGRAMS AND TO LEVERAGE NOT 4437 02:55:40,920 --> 02:55:42,880 ONLY SHARED FINANCIAL RESOURCES 4438 02:55:42,880 --> 02:55:46,920 BUT ALSO RESOURCES SUCH AS 4439 02:55:46,920 --> 02:55:48,240 EXPERTISE AND CLINICAL OR 4440 02:55:48,240 --> 02:55:48,800 SCIENTIFIC NETWORKS. 4441 02:55:48,800 --> 02:55:50,080 I'M GOING TO TELL YOU ABOUT ONE 4442 02:55:50,080 --> 02:55:53,240 OF THE NEW PROGRAMS IN JUST A 4443 02:55:53,240 --> 02:55:54,840 FEW MINUTES. 4444 02:55:54,840 --> 02:55:56,000 FINALLY FUNDING PARTNERSHIPS 4445 02:55:56,000 --> 02:56:00,840 ALLOW US TO BREAK DOWN THE SILOS 4446 02:56:00,840 --> 02:56:02,040 THAT SEPARATE DISEASE SPACES AND 4447 02:56:02,040 --> 02:56:04,080 REPLACE THEM WITH BRIDGES. 4448 02:56:04,080 --> 02:56:05,280 WE'VE USED THESE PARTNERSHIPS TO 4449 02:56:05,280 --> 02:56:08,560 DO JUST THAT AND TO BUILD 4450 02:56:08,560 --> 02:56:12,400 BRIDGES BETWEEN FTD AND OTHER 4451 02:56:12,400 --> 02:56:16,520 NEURODEGENERATIVE DISEASES. 4452 02:56:16,520 --> 02:56:20,200 THIS IS A SNAPSHOT OF CURRENT 4453 02:56:20,200 --> 02:56:20,760 RESEARCH GRANT PORTFOLIO, 4454 02:56:20,760 --> 02:56:23,640 BASICALLY OUR GRANTS FALL INTO 4455 02:56:23,640 --> 02:56:24,480 FOUR CATEGORIES. 4456 02:56:24,480 --> 02:56:27,120 WE HAVE FELLOWSHIPS THAT SUPPORT 4457 02:56:27,120 --> 02:56:28,240 RESEARCHERS WHO ARE STILL 4458 02:56:28,240 --> 02:56:31,080 FINISHING THEIR TRAINING AND NOT 4459 02:56:31,080 --> 02:56:34,120 YET EMBARKING ON AN INDEPENDENT 4460 02:56:34,120 --> 02:56:34,880 RESEARCH PROGRAM. 4461 02:56:34,880 --> 02:56:37,520 WE FUND PILOT GRANTS FOR EARLY 4462 02:56:37,520 --> 02:56:38,200 CAREER RESEARCHERS. 4463 02:56:38,200 --> 02:56:40,920 WE FUND A NUMBER OF PROGRAMS 4464 02:56:40,920 --> 02:56:43,680 THAT SUPPORT BOTH PRE-CLINICAL 4465 02:56:43,680 --> 02:56:46,160 AND CLINICAL DRUG DEVELOPMENT. 4466 02:56:46,160 --> 02:56:48,360 FINALLY PROJECTS AND PROGRAMS 4467 02:56:48,360 --> 02:56:50,920 THAT FOCUS ON BIOMEDICAL 4468 02:56:50,920 --> 02:56:52,400 DISCOVERY AND DEVELOPMENT. 4469 02:56:52,400 --> 02:56:55,400 WITH THE EXCEPTION OF THE PILOT 4470 02:56:55,400 --> 02:56:59,480 GRANT ALL OF OUR OTHER PROGRAMS 4471 02:56:59,480 --> 02:57:01,040 ARE EITHER PARTNERSHIP PROGRAMS 4472 02:57:01,040 --> 02:57:04,080 OR THEY ARE PROGRAMS WHERE WE 4473 02:57:04,080 --> 02:57:06,480 HAVE FUNDED ONE OR MORE TARGETED 4474 02:57:06,480 --> 02:57:09,360 PROJECTS THAT, AGAIN, HAVE 4475 02:57:09,360 --> 02:57:10,280 RELEVANCE FOR BOTH 4476 02:57:10,280 --> 02:57:10,800 ORGANIZATIONS. 4477 02:57:10,800 --> 02:57:13,200 I'M GOING TO GIVE A FEW EXAMPLES 4478 02:57:13,200 --> 02:57:14,280 OF PARTNERSHIP PROGRAMS AND WHAT 4479 02:57:14,280 --> 02:57:15,720 THEY HAVE ALLOWED US TO DO. 4480 02:57:15,720 --> 02:57:17,800 FIRST OF ALL I WANT TO TALK 4481 02:57:17,800 --> 02:57:19,680 ABOUT OUR FELLOWSHIPS. 4482 02:57:19,680 --> 02:57:22,320 WE HAVE GIVEN POSTDOCTORAL 4483 02:57:22,320 --> 02:57:24,720 FELLOWSHIPS SINCE 2009 BUT THE 4484 02:57:24,720 --> 02:57:26,160 ORIGINAL FUNDING MECHANISM 4485 02:57:26,160 --> 02:57:30,400 REALLY DIDN'T GIVE US THE 4486 02:57:30,400 --> 02:57:32,160 OPPORTUNITY TO FOCUS ON SPECIFIC 4487 02:57:32,160 --> 02:57:34,360 NEEDS OF CLINICIAN RESEARCHERS 4488 02:57:34,360 --> 02:57:36,320 WHO FACE SPECIAL CHALLENGES IN 4489 02:57:36,320 --> 02:57:38,400 TERMS OF COST AND ALSO DEVOTING 4490 02:57:38,400 --> 02:57:40,600 TIME TO THEIR RESEARCH AND 4491 02:57:40,600 --> 02:57:45,480 BALANCING THAT WITH THEIR 4492 02:57:45,480 --> 02:57:46,800 CLINICAL RESPONSIBILITIES. 4493 02:57:46,800 --> 02:57:50,920 IN ADDITION WE WANTED TO ENGAGE 4494 02:57:50,920 --> 02:57:53,160 PEOPLE IN FTD RESEARCH EARLY IN 4495 02:57:53,160 --> 02:57:54,960 CLINICAL CAREERS AND BECOME NOT 4496 02:57:54,960 --> 02:57:56,800 ONLY RESEARCHERS BUT ALSO 4497 02:57:56,800 --> 02:57:58,800 AMBASSADORS, TO RAISE AWARENESS 4498 02:57:58,800 --> 02:58:05,440 OF FTD AS ONE OF THE LESS COMMON 4499 02:58:05,440 --> 02:58:07,800 DEMENTIAS, OFTEN MISDIAGNOSED OR 4500 02:58:07,800 --> 02:58:09,040 NOT RECOGNIZED EVEN BY 4501 02:58:09,040 --> 02:58:09,480 SPECIALISTS. 4502 02:58:09,480 --> 02:58:11,600 WE WERE ABLE TO PARTNER WITH THE 4503 02:58:11,600 --> 02:58:13,560 AMERICAN BRAIN FOUNDATION AND 4504 02:58:13,560 --> 02:58:16,160 THROUGH THEIR COLLABORATION WITH 4505 02:58:16,160 --> 02:58:16,720 THE AMERICAN ACADEMY OF 4506 02:58:16,720 --> 02:58:20,000 NEUROLOGY WE WERE ABLE TO FUND 4507 02:58:20,000 --> 02:58:22,960 ONE OF AAN'S CLINICAL RESEARCH 4508 02:58:22,960 --> 02:58:25,080 TRAINING SCHOLARSHIPS 4509 02:58:25,080 --> 02:58:25,920 SPECIFICALLY FOR FTD. 4510 02:58:25,920 --> 02:58:30,080 THE PHOTOS YOU SEE HERE ARE THE 4511 02:58:30,080 --> 02:58:31,280 TWO FIRST RECIPIENTS OF THE 4512 02:58:31,280 --> 02:58:33,240 SCHOLARSHIP AND WE ARE VERY 4513 02:58:33,240 --> 02:58:37,520 HAPPY TO LEARN THAT OUR FIRST 4514 02:58:37,520 --> 02:58:38,720 WINNER, INDIRA GARCIA CORDERO, 4515 02:58:38,720 --> 02:58:40,880 IS GOING TO FULFILL THAT SECOND 4516 02:58:40,880 --> 02:58:42,960 GOAL AND SHE IS PLANNING TO 4517 02:58:42,960 --> 02:58:45,280 RETURN SOON TO HER NATIVE 4518 02:58:45,280 --> 02:58:47,880 ARGENTINA WHERE SHE HOPES TO 4519 02:58:47,880 --> 02:58:49,520 RAISE AWARENESS OF FTD AND 4520 02:58:49,520 --> 02:58:51,720 DEMENTIA IN GENERAL IN LATIN 4521 02:58:51,720 --> 02:58:54,360 AMERICA. 4522 02:58:54,360 --> 02:58:56,240 WE ALSO HAVE A NEW PROGRAM, 4523 02:58:56,240 --> 02:58:57,720 EXAMPLE OF HOW PARTNERSHIPS CAN 4524 02:58:57,720 --> 02:59:00,160 LET YOU DEVELOP NEW INITIATIVES. 4525 02:59:00,160 --> 02:59:03,760 THIS IS A PARTNERSHIP WITH THE 4526 02:59:03,760 --> 02:59:06,520 ALS ASSOCIATION. 4527 02:59:06,520 --> 02:59:08,040 FTD AND ALS WERE FOR A LONG TIME 4528 02:59:08,040 --> 02:59:10,720 THOUGHT TO BE SEPARATE 4529 02:59:10,720 --> 02:59:11,040 DISORDERS. 4530 02:59:11,040 --> 02:59:13,120 WE KNOW THERE ARE MANY 4531 02:59:13,120 --> 02:59:14,800 COMMONALITIES BOTH AT THE 4532 02:59:14,800 --> 02:59:16,640 CLINICAL LEVEL AND ALSO AT THE 4533 02:59:16,640 --> 02:59:18,480 BIOLOGICAL LEVEL. 4534 02:59:18,480 --> 02:59:21,680 SO WE HAVE OVERLAPPING CLINICAL 4535 02:59:21,680 --> 02:59:22,760 PRESENTATIONS. 4536 02:59:22,760 --> 02:59:26,520 SOME INDIVIDUALS WITH ALS 4537 02:59:26,520 --> 02:59:28,480 DISPLAYING THE BEHAVIORAL AND 4538 02:59:28,480 --> 02:59:32,520 LANGUAGE SYMPTOMS OF FTD AND 4539 02:59:32,520 --> 02:59:34,560 VICE VERSE THAT, DISPLAYING 4540 02:59:34,560 --> 02:59:36,000 MOTOR SYMPTOMS OF MOTOR NEURON 4541 02:59:36,000 --> 02:59:36,640 DISEASE. 4542 02:59:36,640 --> 02:59:38,800 THERE ARE ALSO SIMILARITIES IN 4543 02:59:38,800 --> 02:59:41,280 TERMS OF BIOLOGY AT THE 4544 02:59:41,280 --> 02:59:43,760 MOLECULAR AND CELLULAR LEVEL, 4545 02:59:43,760 --> 02:59:46,520 AND, FOR EXAMPLE, THE MUTATION 4546 02:59:46,520 --> 02:59:49,120 OF THE C 9 ORF 72 GENE, THE MOST 4547 02:59:49,120 --> 02:59:50,880 COMMON GENE MUTATION IN FTD IS 4548 02:59:50,880 --> 02:59:53,400 ALSO THE MOST COMMON GENE 4549 02:59:53,400 --> 02:59:55,360 MUTATION IN ALS. 4550 02:59:55,360 --> 02:59:58,880 BUT DESPITE THE SIMILARITIES 4551 02:59:58,880 --> 03:00:00,640 THERE'S NOT A CLINICAL 4552 03:00:00,640 --> 03:00:02,600 ASSESSMENT MEASURE USED BROADLY 4553 03:00:02,600 --> 03:00:07,080 ACROSS THE ENTIRE FTD/ALS 4554 03:00:07,080 --> 03:00:07,560 SPECTRUM. 4555 03:00:07,560 --> 03:00:09,800 ALS AND FTD ALSO PRESENT 4556 03:00:09,800 --> 03:00:12,400 CHALLENGES IN TERMS OF CLINICAL 4557 03:00:12,400 --> 03:00:14,320 CARE AND RUNNING CLINICAL TRIALS 4558 03:00:14,320 --> 03:00:16,920 BECAUSE THEY ARE DEVASTATING 4559 03:00:16,920 --> 03:00:18,240 DISORDERS WHERE PEOPLE ARE 4560 03:00:18,240 --> 03:00:20,640 CHALLENGED TO GET BACK AND FORTH 4561 03:00:20,640 --> 03:00:22,720 TO THE CLINIC AND WHERE THEIR 4562 03:00:22,720 --> 03:00:23,960 BEHAVIOR IN PARTICULAR IN THE 4563 03:00:23,960 --> 03:00:27,040 CLINIC IS OFTEN NOT THE BEHAVIOR 4564 03:00:27,040 --> 03:00:28,760 YOU SEE AT HOME. 4565 03:00:28,760 --> 03:00:30,920 DIGITAL TOOLS OFFER WAYS TO GET 4566 03:00:30,920 --> 03:00:33,120 AROUND THOSE PROBLEMS BUT 4567 03:00:33,120 --> 03:00:34,560 DESPITE A PLETHORA OF DIGITAL 4568 03:00:34,560 --> 03:00:36,640 TOOLS MAKING IT THROUGH THE 4569 03:00:36,640 --> 03:00:38,800 PILOT STAGE VERY FEW HAVE 4570 03:00:38,800 --> 03:00:42,120 CONTINUED TO ADVANCE ALONG THE 4571 03:00:42,120 --> 03:00:43,560 PIPELINE TOWARDS VALIDATION. 4572 03:00:43,560 --> 03:00:46,040 THIS PROGRAM AIMS TO ADDRESS ALL 4573 03:00:46,040 --> 03:00:47,000 OF THOSE NEEDS. 4574 03:00:47,000 --> 03:00:50,400 AND SO IT WILL ADVANCE THE 4575 03:00:50,400 --> 03:00:51,520 FURTHER DEVELOPMENT OF DIGITAL 4576 03:00:51,520 --> 03:00:53,920 TOOLS AT THE INTERSECTION 4577 03:00:53,920 --> 03:00:56,080 BETWEEN ALS AND FTD, AND IT WILL 4578 03:00:56,080 --> 03:00:59,360 SPECIFICALLY ASK THAT THE TOOLS 4579 03:00:59,360 --> 03:01:00,280 BEING TESTED ALREADY HAVE 4580 03:01:00,280 --> 03:01:01,800 PRELIMINARY DATA AND ARE READY 4581 03:01:01,800 --> 03:01:06,720 TO ADVANCE BEYOND THE PILOT 4582 03:01:06,720 --> 03:01:07,560 STAGE. 4583 03:01:07,560 --> 03:01:08,520 FOR THE THIRD EXAMPLE I'D LIKE 4584 03:01:08,520 --> 03:01:10,760 TO TALK A LITTLE BIT ABOUT OUR 4585 03:01:10,760 --> 03:01:12,960 PROGRAMS THAT WE FUND IN 4586 03:01:12,960 --> 03:01:14,600 PARTNERSHIP WITH THE ALZHEIMER'S 4587 03:01:14,600 --> 03:01:19,640 DRUG DISCOVERY FOUNDATION. 4588 03:01:19,640 --> 03:01:21,200 WE HAVE THESE PROGRAMS LOOKING 4589 03:01:21,200 --> 03:01:24,200 AT PRE-CLINICAL AND CLINICAL 4590 03:01:24,200 --> 03:01:26,840 LEVEL. 4591 03:01:26,840 --> 03:01:30,120 ACCELERATING DRUG DISCOVERY FOR 4592 03:01:30,120 --> 03:01:33,080 FTZ OUR LONGEST RUNNING 4593 03:01:33,080 --> 03:01:34,400 PARTNERSHIP PROGRAM, DISCOVERED 4594 03:01:34,400 --> 03:01:36,680 DRUG DISCOVERY AND PRE-CLINICAL 4595 03:01:36,680 --> 03:01:42,160 RESEARCH THAT NOW FOCUSES 4596 03:01:42,160 --> 03:01:44,120 SUPPORTING FURTHER DEVELOPMENT 4597 03:01:44,120 --> 03:01:47,520 OF LEAD COMPOUNDS THROUGH 4598 03:01:47,520 --> 03:01:48,840 SUPPORT FOR LEAD OPTIMIZATION, 4599 03:01:48,840 --> 03:01:51,640 IN VIVO EFFICACY STUDIES IN 4600 03:01:51,640 --> 03:01:51,880 ANIMALS. 4601 03:01:51,880 --> 03:01:56,240 WE WERE ABLE TO BUILD ON THAT 4602 03:01:56,240 --> 03:01:58,360 PARTNERSHIP TO FUND EARLY STAGE 4603 03:01:58,360 --> 03:02:00,760 CLINICAL TRIALS SINCE 2016. 4604 03:02:00,760 --> 03:02:03,040 AND WE CURRENTLY HAVE FOUR SUCH 4605 03:02:03,040 --> 03:02:05,560 ACTIVE TRIALS AND THEY ARE 4606 03:02:05,560 --> 03:02:11,160 LOOKING AT BOTH NOVEL AGENTS AND 4607 03:02:11,160 --> 03:02:16,400 ALSO REPURPOSED DRUGS. 4608 03:02:16,400 --> 03:02:18,600 4609 03:02:18,600 --> 03:02:21,760 SINCE 2015, OUR PARTNERSHIP 4610 03:02:21,760 --> 03:02:23,760 PROGRAMS HAVE AWARDED OVER $20 4611 03:02:23,760 --> 03:02:25,240 MILLION ACROSS SIX BRANT 4612 03:02:25,240 --> 03:02:27,160 PROGRAMS AND THESE ARRANGEMENTS 4613 03:02:27,160 --> 03:02:29,200 ALLOWED US TO ACHIEVE FUNDING 4614 03:02:29,200 --> 03:02:32,920 GOALS SUCH AS SUPPORTING 4615 03:02:32,920 --> 03:02:33,920 CLINICIAN RESEARCHERS, TO 4616 03:02:33,920 --> 03:02:35,360 LEVERAGE RESOURCES, AGAIN NOT 4617 03:02:35,360 --> 03:02:41,680 JUST TIME BUT ALSO EXPERTISE, 4618 03:02:41,680 --> 03:02:43,560 AND RESEARCH NETWORKS TO ALIGN 4619 03:02:43,560 --> 03:02:45,320 RESEARCH INTERESTS ACROSS 4620 03:02:45,320 --> 03:02:47,400 DISEASE SPACES, AND IMPORTANTLY 4621 03:02:47,400 --> 03:02:50,120 TO BUILD STRONG AND PRODUCTIVE 4622 03:02:50,120 --> 03:02:51,320 RELATIONSHIPS WITH OTHER 4623 03:02:51,320 --> 03:02:52,560 NON-PROFITS THAT WE HOPE WE WILL 4624 03:02:52,560 --> 03:02:56,760 BE ABLE TO BUILD ON TO OFFER 4625 03:02:56,760 --> 03:02:58,000 ADDITIONAL SUCH PROGRAMS IN THE 4626 03:02:58,000 --> 03:02:58,480 FUTURE. 4627 03:02:58,480 --> 03:02:59,880 BEFORE I SIT DOWN I WANT TO 4628 03:02:59,880 --> 03:03:02,040 THANK ALL OF YOU WHO TOLD YOUR 4629 03:03:02,040 --> 03:03:04,240 STORIES TODAY. 4630 03:03:04,240 --> 03:03:05,880 THEY WERE INCREDIBLY COMPELLING. 4631 03:03:05,880 --> 03:03:08,640 AND THEY ARE A REMINDER OF WHY 4632 03:03:08,640 --> 03:03:10,680 WE FUND RESEARCH AND AN 4633 03:03:10,680 --> 03:03:12,280 INSPIRATION FOR OUR RESEARCH 4634 03:03:12,280 --> 03:03:12,520 COMMUNITY. 4635 03:03:12,520 --> 03:03:20,440 THANK YOU. 4636 03:03:20,440 --> 03:03:25,720 >> NEXT UP MANNISH RAISINGHANI 4637 03:03:25,720 --> 03:03:27,480 DELIVERING HIS TALK VIRTUALLY. 4638 03:03:27,480 --> 03:03:32,480 >> AN INTRODUCTION, TARGET ALS 4639 03:03:32,480 --> 03:03:35,360 FOUNDATION, WE'RE IN EXISTENCE 4640 03:03:35,360 --> 03:03:37,240 SINCE 2013 TO FINDING EFFECTIVE 4641 03:03:37,240 --> 03:03:41,920 TREATMENT FOR ALS. 4642 03:03:41,920 --> 03:03:45,320 WE ARE A BIOMEDICAL RESEARCH 4643 03:03:45,320 --> 03:03:46,480 FOUNDATION, WHAT DIFFERENTIATED 4644 03:03:46,480 --> 03:03:48,640 US IS APPROACH WE'VE TAKEN TO 4645 03:03:48,640 --> 03:03:49,400 FUNDING ALS RESEARCH. 4646 03:03:49,400 --> 03:03:51,320 WE FEEL THERE ARE LESSONS THAT 4647 03:03:51,320 --> 03:03:53,240 MAY BE APPLICABLE TO OTHER 4648 03:03:53,240 --> 03:03:55,080 DISEASE AREAS AS WELL. 4649 03:03:55,080 --> 03:03:57,600 WHEN WE STARTED IN 2013 THE 4650 03:03:57,600 --> 03:03:59,480 FIRST TASK TO UNDERSTAND WHAT 4651 03:03:59,480 --> 03:04:01,120 WERE KEY BARRIERS STANDING IN 4652 03:04:01,120 --> 03:04:05,400 THE WAY OF ALS RESEARCH AND DRUG 4653 03:04:05,400 --> 03:04:05,640 DISCOVERY. 4654 03:04:05,640 --> 03:04:07,120 IT WAS DISCOVERED 150 YEARS AGO, 4655 03:04:07,120 --> 03:04:08,640 PROGRESS WAS SLOW AND ITERATIVE. 4656 03:04:08,640 --> 03:04:10,840 WHAT WE FOUND WHEN WE STARTED 4657 03:04:10,840 --> 03:04:12,280 WAS THAT THERE WERE SIX KEY 4658 03:04:12,280 --> 03:04:16,480 BARRIERS STANDING IN THE WAY OF 4659 03:04:16,480 --> 03:04:16,760 PROGRESS. 4660 03:04:16,760 --> 03:04:20,280 THEY ARE LISTED ON THE SLIDE 4661 03:04:20,280 --> 03:04:20,480 HERE. 4662 03:04:20,480 --> 03:04:23,040 SOME OF THESE YOU'LL FIND TO BE 4663 03:04:23,040 --> 03:04:26,120 FAMILIAR, NOT NECESSARILY UNIQUE 4664 03:04:26,120 --> 03:04:28,280 TO ALS RESEARCH COMMUNITY. 4665 03:04:28,280 --> 03:04:32,000 BUT WHAT HAPPENS IS THESE ARE 4666 03:04:32,000 --> 03:04:34,520 BARRIERS THAT ARE FACED BY ALL 4667 03:04:34,520 --> 03:04:37,040 DIFFERENT EFFORTS ON DIFFERENT 4668 03:04:37,040 --> 03:04:38,240 DISEASE AREAS, BUT PARTICULARLY 4669 03:04:38,240 --> 03:04:40,440 ACUTE WHEN IT COMES TO SMALLER 4670 03:04:40,440 --> 03:04:41,320 RESEARCH COMMUNITIES. 4671 03:04:41,320 --> 03:04:44,400 SO WHAT WE FOUND THERE WAS A 4672 03:04:44,400 --> 03:04:46,440 REAL GAP IN FUNDING FOR 4673 03:04:46,440 --> 03:04:47,480 TRANSLATIONAL RESEARCH AND 4674 03:04:47,480 --> 03:04:49,400 INCENTIVES FOR PEOPLE TO WORK 4675 03:04:49,400 --> 03:04:50,280 TOGETHER COLLABORATIVELY. 4676 03:04:50,280 --> 03:04:55,200 WHAT WE ALSO FOUND WAS THAT THE 4677 03:04:55,200 --> 03:04:56,400 APPROACH TO RESEARCH, WITHIN 4678 03:04:56,400 --> 03:04:58,720 ACADEMIA, MORE SO WHEN IT CAMES 4679 03:04:58,720 --> 03:04:59,720 TO ACADEMICS, GROUPS WORKING 4680 03:04:59,720 --> 03:04:59,960 TOGETHER. 4681 03:04:59,960 --> 03:05:01,880 WE ALSO FOUND THERE WAS A REAL 4682 03:05:01,880 --> 03:05:05,560 BARRIER TO WORKING ON ALS 4683 03:05:05,560 --> 03:05:08,880 BECAUSE OF CHALLENGES IN 4684 03:05:08,880 --> 03:05:11,200 ACCESSION AN ANTIBODY TO ANIMAL 4685 03:05:11,200 --> 03:05:16,320 MODELS TO PRECIOUS HUMAN B 4686 03:05:16,320 --> 03:05:16,640 IOSPECIMENS. 4687 03:05:16,640 --> 03:05:18,520 THERE WAS LIMITED INVESTMENT IN 4688 03:05:18,520 --> 03:05:20,440 ALS RESEARCH. 4689 03:05:20,440 --> 03:05:22,680 ANOTHER KEY ISSUE WAS AS YOU 4690 03:05:22,680 --> 03:05:24,440 KNOW WE TYPICALLY RELY ON PEER 4691 03:05:24,440 --> 03:05:28,800 REVIEW PROCESS TO MAKE FUNDING 4692 03:05:28,800 --> 03:05:29,160 DECISIONS. 4693 03:05:29,160 --> 03:05:31,640 THEY WORK WELL BUT HAVE 4694 03:05:31,640 --> 03:05:32,720 CHALLENGES, EXACERBATED WITH 4695 03:05:32,720 --> 03:05:33,320 SMALLER RESEARCH COMMUNITIES 4696 03:05:33,320 --> 03:05:34,600 BECAUSE OF THE SAME PEOPLE GOING 4697 03:05:34,600 --> 03:05:38,120 TO ACCESS FUNDING ARE MAKING 4698 03:05:38,120 --> 03:05:45,120 DECISIONS, THERE'S A POTENTIAL 4699 03:05:45,120 --> 03:05:46,360 FOR RESOURCES (INDISCERNIBLE). 4700 03:05:46,360 --> 03:05:56,720 SEVERAL ISSUES WITH LICENSING 4701 03:05:56,720 --> 03:05:57,720 AGREEMENTS, DISCOURAGING 4702 03:05:57,720 --> 03:05:59,360 PARTICIPATION FROM TECH AND 4703 03:05:59,360 --> 03:06:01,800 RESEARCH COMMUNITIES. 4704 03:06:01,800 --> 03:06:04,880 THE APPROACH WAS TO CREATE 4705 03:06:04,880 --> 03:06:06,720 ECOSYSTEMS WITH SIX STRATEGIES 4706 03:06:06,720 --> 03:06:08,960 SHOWN HERE MUTUALLY REINFORCING, 4707 03:06:08,960 --> 03:06:11,000 MEANT TO ADDRESS BARRIERS THAT 4708 03:06:11,000 --> 03:06:13,320 WE IDENTIFIED. 4709 03:06:13,320 --> 03:06:18,440 THE FIRST WAS EXCLUSIVELY FUND 4710 03:06:18,440 --> 03:06:19,000 COLLABORATIVE PROJECTS, 4711 03:06:19,000 --> 03:06:20,400 SCIENTISTS HAVE TO COME 4712 03:06:20,400 --> 03:06:23,520 TOGETHER, WORK ON THE SAME IDEA 4713 03:06:23,520 --> 03:06:23,800 TOGETHER. 4714 03:06:23,800 --> 03:06:26,040 MOVE THE IDEA FORWARD OR COME TO 4715 03:06:26,040 --> 03:06:28,440 A GO/NO-GO DECISIONS. 4716 03:06:28,440 --> 03:06:31,920 WE IN SOME FUNDING OPPORTUNITIES 4717 03:06:31,920 --> 03:06:32,920 REQUIRED OPEN-ENDED 4718 03:06:32,920 --> 03:06:37,400 COLLABORATION, ONLY ACADEMICS, 4719 03:06:37,400 --> 03:06:39,080 BUT IN SOME FUNDING REQUIRED 4720 03:06:39,080 --> 03:06:41,120 BIOTECH BE A COLLABORATOR. 4721 03:06:41,120 --> 03:06:45,720 WE WANTED TO DEMOCRATIZE ALS 4722 03:06:45,720 --> 03:06:50,840 RESEARCH AND LOWER BARRIERS, 4723 03:06:50,840 --> 03:06:52,800 CREATING CORE FACILITIES THAT 4724 03:06:52,800 --> 03:06:54,480 CREATE, COLLECT, PROVIDE ACCESS 4725 03:06:54,480 --> 03:06:56,040 TO SCIENTIFIC TOOLS AND 4726 03:06:56,040 --> 03:06:59,840 RESOURCES, ANTIBODIES TO HUMAN 4727 03:06:59,840 --> 03:07:04,480 STEM CELLS, GENOMIC DATASETS TO 4728 03:07:04,480 --> 03:07:05,360 BIOFLUID SAMPLES, ALLOWED ANYONE 4729 03:07:05,360 --> 03:07:09,960 IN THE WORLD TO WORK ON THIS 4730 03:07:09,960 --> 03:07:11,160 WITHOUT ENTER IN COLLABORATION, 4731 03:07:11,160 --> 03:07:17,280 ONLY TO ACCESS THESE TOOLS. 4732 03:07:17,280 --> 03:07:24,320 THE THIRD THING, WHEN WE 4733 03:07:24,320 --> 03:07:27,360 LAUNCHED, WE LAUNCHED 4734 03:07:27,360 --> 03:07:29,320 INITIATIVE, TOOK THEM ALONG, WE 4735 03:07:29,320 --> 03:07:32,040 ALSO HAVE REPRESENTATION OF 4736 03:07:32,040 --> 03:07:33,480 PHARMA BIOTECH ON BOARDS, REVIEW 4737 03:07:33,480 --> 03:07:34,680 COMMITTEE, HIRED FROM THE 4738 03:07:34,680 --> 03:07:37,880 INDUSTRY AND ADVISORS AS WELL. 4739 03:07:37,880 --> 03:07:42,320 TO ENSURE WE HAVE A CONFLICT OF 4740 03:07:42,320 --> 03:07:44,200 INTEREST FREE AND PROCESS FOR 4741 03:07:44,200 --> 03:07:45,720 REVIEW OF FUNDING DECISIONS, 4742 03:07:45,720 --> 03:07:47,600 USING PEER REVIEW HAVE AN 4743 03:07:47,600 --> 03:07:50,360 INDEPENDENT REVIEW COMMITTEE. 4744 03:07:50,360 --> 03:07:58,200 BY DESIGN THIS COMMITTEE IS 50% 4745 03:07:58,200 --> 03:08:01,400 FROM ACADEMIA, NON-PROFIT, 50% 4746 03:08:01,400 --> 03:08:02,360 FROM CAPITAL. 4747 03:08:02,360 --> 03:08:04,320 REFLECTING THE NEED OF ALS 4748 03:08:04,320 --> 03:08:04,720 RESEARCH. 4749 03:08:04,720 --> 03:08:08,160 VERY FEW FROM THE COMMUNITY 4750 03:08:08,160 --> 03:08:09,720 ITSELF BECAUSE WE WANT EVERYONE 4751 03:08:09,720 --> 03:08:11,040 TO APPLY. 4752 03:08:11,040 --> 03:08:18,560 MEMBERS CAN APPLY FOR OR RECEIVE 4753 03:08:18,560 --> 03:08:19,960 TARGETING ALS FUNDING, THIS HAS 4754 03:08:19,960 --> 03:08:22,200 GIVEN A POSITION OF CREDIBILITY. 4755 03:08:22,200 --> 03:08:29,200 ANYONE CAN APPLY AND GET FUNDING 4756 03:08:29,200 --> 03:08:30,080 IRRESPECTIVE WITH BOARD MEMBERS 4757 03:08:30,080 --> 03:08:34,640 OR ANYONE ELSE. 4758 03:08:34,640 --> 03:08:38,040 A KEY PRINCIPAL HAS BEEN FUNDING 4759 03:08:38,040 --> 03:08:39,720 ALLOWS INVESTIGATORS IN ACADEMIA 4760 03:08:39,720 --> 03:08:40,920 OR PHARMA BIOTECH TO RETAIN 4761 03:08:40,920 --> 03:08:42,240 RIGHTS TO THEIR DATA AND 4762 03:08:42,240 --> 03:08:42,880 INTELLECTUAL PROPERTY. 4763 03:08:42,880 --> 03:08:45,280 WE WANT NOTHING IN RETURN. 4764 03:08:45,280 --> 03:08:47,720 THIS IS IMPORTANT GETTING 4765 03:08:47,720 --> 03:08:50,040 VENTURE CAPITAL AND BIOTECH 4766 03:08:50,040 --> 03:08:51,360 INVOLVED, GETTING LAUNCHED. 4767 03:08:51,360 --> 03:08:54,400 LASTLY A HUB OF NETWORKING AND 4768 03:08:54,400 --> 03:08:55,120 COMMUNICATION FOR RESEARCH 4769 03:08:55,120 --> 03:08:57,080 COMMUNITY WORLDWIDE. 4770 03:08:57,080 --> 03:08:58,720 WE SPEAK TO ACADEMICS, 4771 03:08:58,720 --> 03:08:59,560 NON-PROFITS, VENTURE CAPITAL, 4772 03:08:59,560 --> 03:09:01,200 CONNECT THEM BASED ON 4773 03:09:01,200 --> 03:09:01,800 OVERLAPPING INTEREST AND MOVE 4774 03:09:01,800 --> 03:09:04,760 OUT OF THE WAY. 4775 03:09:04,760 --> 03:09:08,200 THE IDEA IS TO FACILITATE, NOT 4776 03:09:08,200 --> 03:09:09,120 GAIN CREDIT. 4777 03:09:09,120 --> 03:09:11,080 THESE HAVE ALLOWED US TO IMPACT 4778 03:09:11,080 --> 03:09:14,040 RESEARCH IN A SIGNIFICANT WAY. 4779 03:09:14,040 --> 03:09:15,680 I'M GOING TO ILLUSTRATE A POINT 4780 03:09:15,680 --> 03:09:20,480 ABOUT THESE PROJECTS WE FUND. 4781 03:09:20,480 --> 03:09:21,280 TYPICALLY IT MEANS INVESTIGATORS 4782 03:09:21,280 --> 03:09:23,560 COME TOGETHER WORKING ON THEIR 4783 03:09:23,560 --> 03:09:24,760 OWN IDEAS, FUNDING FROM A COMMON 4784 03:09:24,760 --> 03:09:27,200 POOL, THEY WORK IN THEIR OWN 4785 03:09:27,200 --> 03:09:27,520 LABS. 4786 03:09:27,520 --> 03:09:30,280 IN OUR CASE WE REQUIRE BY 4787 03:09:30,280 --> 03:09:33,120 DEFINITION SCIENTISTS HAVE TO 4788 03:09:33,120 --> 03:09:34,000 SHOW COMPLEMENTARY EXPERTISE, 4789 03:09:34,000 --> 03:09:37,520 ALL THE PROJECTS WE FUND BY 4790 03:09:37,520 --> 03:09:38,280 DESIGN ARE MULTI-DISCIPLINARY, 4791 03:09:38,280 --> 03:09:40,920 SHOWN ON THE RIGHT. 4792 03:09:40,920 --> 03:09:43,440 OF 59 PROJECTS, FOUR TO SIX 4793 03:09:43,440 --> 03:09:46,040 COLLABORATORS, 60% HAD A PHARMA 4794 03:09:46,040 --> 03:09:48,880 BIOTECH BASED SCIENTIST LIED THE 4795 03:09:48,880 --> 03:09:51,520 PROJECT, HAVE REPRESENTED 4796 03:09:51,520 --> 03:09:55,040 SCIENTISTS FROM 32 COMPANIES, 60 4797 03:09:55,040 --> 03:09:58,760 ACADEMIC INSTITUTIONS WORLDWIDE. 4798 03:09:58,760 --> 03:10:03,320 WE HAVE CREATED ACCESS TO 4799 03:10:03,320 --> 03:10:04,920 RESEARCH TOOLS AND RESOURCES. 4800 03:10:04,920 --> 03:10:06,160 BY PROVIDING TOOLS WE KNOW THEY 4801 03:10:06,160 --> 03:10:08,560 HAVE BEEN USED BY OVER 975 4802 03:10:08,560 --> 03:10:09,960 PROJECTS WORLDWIDE NOW. 4803 03:10:09,960 --> 03:10:14,000 THIS IS A QUANTITATIVE IMPACT 4804 03:10:14,000 --> 03:10:14,800 BUT THERE'S A COLLABORATIVE 4805 03:10:14,800 --> 03:10:15,760 IMPACT. 4806 03:10:15,760 --> 03:10:21,200 IF IT WASN'T FOR ACCESS TO TOOL 4807 03:10:21,200 --> 03:10:24,080 THEY MAY NOT HAVE GOTTEN DATA 4808 03:10:24,080 --> 03:10:25,600 USED FOR R01 GRANTS TO BE 4809 03:10:25,600 --> 03:10:25,920 SUCCESSFUL. 4810 03:10:25,920 --> 03:10:29,520 IN TERMS OF IMPACT WE'VE SEEN 4811 03:10:29,520 --> 03:10:31,200 USING THESE STRATEGIES, 60% OF 4812 03:10:31,200 --> 03:10:39,520 COLLABORATIVE PROJECTS HAVE LED 4813 03:10:39,520 --> 03:10:49,840 TOEN ON GOING DRUG. 4814 03:10:51,480 --> 03:10:53,920 WE CALL THIS A 7-PILLAR 4815 03:10:53,920 --> 03:10:54,600 STRATEGY. 4816 03:10:54,600 --> 03:11:01,920 WE KNOW AS SCIENCE EVOLVES WE'RE 4817 03:11:01,920 --> 03:11:04,240 KEEN ON DIVERSIFYING, LOOKING AT 4818 03:11:04,240 --> 03:11:06,640 WHICH DISCIPLINES TO ENGAGE IN, 4819 03:11:06,640 --> 03:11:08,480 PARTNER WITH ORGANIZATIONS 4820 03:11:08,480 --> 03:11:09,560 WORKING ON RELATED 4821 03:11:09,560 --> 03:11:11,880 NEURODEGENERATIVE DISEASES TO 4822 03:11:11,880 --> 03:11:15,040 BRING IN EXPERTISE AS WELL. 4823 03:11:15,040 --> 03:11:17,920 WE'LL MAINTAIN AND EXPAND TO 4824 03:11:17,920 --> 03:11:20,000 PROVIDE CRITICAL RESEARCH TOOLS. 4825 03:11:20,000 --> 03:11:21,080 WE PARTNER WITH OTHER DISEASE 4826 03:11:21,080 --> 03:11:24,480 ORGANIZATIONS TO BRING IN TALENT 4827 03:11:24,480 --> 03:11:25,120 AND EXPERTISE. 4828 03:11:25,120 --> 03:11:27,320 THE CORE OF THE STRATEGY TO 4829 03:11:27,320 --> 03:11:31,360 DISCOVER AND DEVELOP NEW 4830 03:11:31,360 --> 03:11:36,080 TREATMENTS, TOOLKIT OF 4831 03:11:36,080 --> 03:11:37,920 BIOMARKERS BECAUSE ALS DOES NOT 4832 03:11:37,920 --> 03:11:38,480 HAVE ONE. 4833 03:11:38,480 --> 03:11:41,760 WE WANT TO TREAT ALL FORMS, BUT 4834 03:11:41,760 --> 03:11:42,400 PARTNERING WITH OTHER 4835 03:11:42,400 --> 03:11:43,640 ORGANIZATIONS AND GROW OUR 4836 03:11:43,640 --> 03:11:45,360 ORGANIZATION AS WE MOVE FORWARD. 4837 03:11:45,360 --> 03:11:48,880 I'LL STOP AND THANK YOU FOR YOUR 4838 03:11:48,880 --> 03:11:50,840 ATTENTION AND LATER BE HAPPY TO 4839 03:11:50,840 --> 03:11:57,640 ANSWER ANY QUESTIONS YOU HAVE. 4840 03:11:57,640 --> 03:12:03,560 WE'RE GOING TO OUR NEXT SPEAKER 4841 03:12:03,560 --> 03:12:10,440 AND HAVE A GROUP PANEL 4842 03:12:10,440 --> 03:12:10,720 DISCUSSION. 4843 03:12:10,720 --> 03:12:12,120 VICKY CATTANI, PARKINSON'S 4844 03:12:12,120 --> 03:12:14,840 FOUNDATION, IS NEXT UP. 4845 03:12:14,840 --> 03:12:22,360 4846 03:12:22,360 --> 03:12:29,840 4847 03:12:29,840 --> 03:12:31,440 >> THANK YOU FOR INVITING THE 4848 03:12:31,440 --> 03:12:32,880 FOUNDATION AND ME. 4849 03:12:32,880 --> 03:12:34,720 I WANT TO ECHO THE COMMENTS, 4850 03:12:34,720 --> 03:12:38,680 THANK YOU TO EVERYONE WHO SHARED 4851 03:12:38,680 --> 03:12:41,280 YOUR STORIES. 4852 03:12:41,280 --> 03:12:43,920 I'M ASSOCIATE DIRECTOR OF 4853 03:12:43,920 --> 03:12:47,400 RESEARCH PROGRAMS AT THE 4854 03:12:47,400 --> 03:12:48,960 PARKINSON'S FOUNDATION. 4855 03:12:48,960 --> 03:12:51,600 OUR MISSION IS TO MAKE LIFE 4856 03:12:51,600 --> 03:12:54,040 BETTER FOR PEOPLE WITH 4857 03:12:54,040 --> 03:13:00,040 PARKINSON'S BY IMPROVING CARE 4858 03:13:00,040 --> 03:13:03,560 AND ADVANCING RESEARCH TOWARD A 4859 03:13:03,560 --> 03:13:04,080 CARE. 4860 03:13:04,080 --> 03:13:05,440 OUR FOUNDATION FOCUS IS VERY 4861 03:13:05,440 --> 03:13:06,400 SPECIFIC. 4862 03:13:06,400 --> 03:13:08,480 THAT'S WHY THE NAME IS 4863 03:13:08,480 --> 03:13:09,520 PARKINSON'S FOUNDATION. 4864 03:13:09,520 --> 03:13:11,880 AND THAT IS, YOU KNOW, CONTRARY 4865 03:13:11,880 --> 03:13:19,320 TO KIND OF WHAT NINDS DOES, 4866 03:13:19,320 --> 03:13:21,520 DIVERSE PORTFOLIO OF 4867 03:13:21,520 --> 03:13:21,880 NEUROSCIENCE. 4868 03:13:21,880 --> 03:13:27,640 WE HAVE A FORECAST USED AND 4869 03:13:27,640 --> 03:13:28,960 RESEARCH AREA. 4870 03:13:28,960 --> 03:13:30,360 WE INCENTIVIZE SCIENTISTS TO 4871 03:13:30,360 --> 03:13:32,000 DEVOTE CAREERS TO PARKINSON'S 4872 03:13:32,000 --> 03:13:33,240 DISEASE RESEARCH. 4873 03:13:33,240 --> 03:13:36,200 WE WANT THEM TO KICK START THE 4874 03:13:36,200 --> 03:13:38,040 INNOVATIVE IDEAS OF NEW 4875 03:13:38,040 --> 03:13:41,000 SCIENTISTS AND GRANTS, 4876 03:13:41,000 --> 03:13:42,080 FELLOWSHIPS, PARTNERSHIPS. 4877 03:13:42,080 --> 03:13:43,960 PROVIDE SCIENTISTS WITH FUNDING 4878 03:13:43,960 --> 03:13:49,640 SO THEIR PROMISING IDEAS ARE 4879 03:13:49,640 --> 03:13:49,960 EXPLORED. 4880 03:13:49,960 --> 03:13:52,280 WE DO THAT TO FELLOWSHIP, 4881 03:13:52,280 --> 03:13:53,920 GRANTS, AWARDS. 4882 03:13:53,920 --> 03:13:55,440 I WANT TO FOCUS ON THESE TYPES 4883 03:13:55,440 --> 03:13:56,200 OF AWARDS. 4884 03:13:56,200 --> 03:13:59,080 WE HAVE OTHERS WE CAN DISCUSS 4885 03:13:59,080 --> 03:14:00,360 ONE ON ONE LATER. 4886 03:14:00,360 --> 03:14:03,400 AND I WANT TO REALLY FOCUS ON 4887 03:14:03,400 --> 03:14:08,800 THESE BECAUSE IN PARTICULAR THE 4888 03:14:08,800 --> 03:14:10,320 EARLY CAREER INVESTIGATORS, AND 4889 03:14:10,320 --> 03:14:15,360 LAUNCH AWARDS ON THE BOTTOM IN 4890 03:14:15,360 --> 03:14:20,960 YELLOW ARE REALLY -- THEY REALLY 4891 03:14:20,960 --> 03:14:23,120 COMPLEMENT EACH OTHER FOR 4892 03:14:23,120 --> 03:14:25,880 INVESTIGATORS TO CONVINCE THEM 4893 03:14:25,880 --> 03:14:28,160 TO REALLY STAY IN PARKINSON'S. 4894 03:14:28,160 --> 03:14:29,720 BECAUSE THEN THEY GET THIS 4895 03:14:29,720 --> 03:14:33,160 FUNDING AND THEN THEY GET VERY 4896 03:14:33,160 --> 03:14:35,040 KEY DATA THAT THEY CAN THEN 4897 03:14:35,040 --> 03:14:39,600 LEVERAGE TO GET THE FIRST R01. 4898 03:14:39,600 --> 03:14:41,640 AND WE SAY CRITICAL BECAUSE A 4899 03:14:41,640 --> 03:14:43,560 LOT OF THESE SCIENTISTS MAY 4900 03:14:43,560 --> 03:14:46,920 START IN PARKINSON'S BUT THEN 4901 03:14:46,920 --> 03:14:49,760 THEY SAY, WELL, YOU KNOW, I 4902 03:14:49,760 --> 03:14:51,200 APPLY FOR R01S BUT DON'T GET 4903 03:14:51,200 --> 03:14:53,920 THEM, I'M GOING TO SWITCH FOR 4904 03:14:53,920 --> 03:14:55,120 ALZHEIMER'S WHICH MIGHT GET 4905 03:14:55,120 --> 03:14:55,360 FUNDED. 4906 03:14:55,360 --> 03:14:57,440 WE WANT THEM TO STAY IN 4907 03:14:57,440 --> 03:14:59,840 PARKINSON'S BECAUSE IT'S A VERY 4908 03:14:59,840 --> 03:15:00,640 COMPLEX DISEASE. 4909 03:15:00,640 --> 03:15:10,000 AND WE DON'T HAVE A CURE. 4910 03:15:10,000 --> 03:15:13,720 WE CAN DOUBLE THE AMOUNT OF 4911 03:15:13,720 --> 03:15:16,880 POSTDOCS DOING PARKINSON'S 4912 03:15:16,880 --> 03:15:17,440 DISEASE RESEARCH. 4913 03:15:17,440 --> 03:15:19,200 IF YOU LEARN HOW TO USE NIH 4914 03:15:19,200 --> 03:15:23,760 REPORTER AND LOOK FOR NUMBER OF 4915 03:15:23,760 --> 03:15:24,480 POSTDOCTORAL FELLOWSHIPS, LOOK 4916 03:15:24,480 --> 03:15:26,680 AT THE NUMBER OF AWARDS THAT 4917 03:15:26,680 --> 03:15:31,320 HAVE BEEN GIVEN TO POSTDOCS OR 4918 03:15:31,320 --> 03:15:32,920 PRE-DOCS STUDYING PARKINSON'S IN 4919 03:15:32,920 --> 03:15:34,880 THE LAB A FEW YEARS YOU CAN SEE 4920 03:15:34,880 --> 03:15:37,400 NUMBERS ARE NOT THAT HIGH, NOT 4921 03:15:37,400 --> 03:15:39,360 THAT MANY YOUNG SCIENTISTS DOING 4922 03:15:39,360 --> 03:15:41,360 POSTDOCTORAL RESEARCH ON 4923 03:15:41,360 --> 03:15:42,680 PARKINSON'S DISEASE. 4924 03:15:42,680 --> 03:15:46,040 WE DON'T GIVE THAT MANY 4925 03:15:46,040 --> 03:15:48,800 FELLOWSHIPS, BUT EVEN IF WE GIVE 4926 03:15:48,800 --> 03:15:51,920 FIVE OR SIX EVERY YEAR WE'RE 4927 03:15:51,920 --> 03:15:55,200 DOUBLING THE NUMBER OF FELLOWS 4928 03:15:55,200 --> 03:15:56,720 STUDYING PARKINSON'S DISEASE. 4929 03:15:56,720 --> 03:16:00,760 WHICH WE THINK IS GREAT. 4930 03:16:00,760 --> 03:16:03,720 SO, OUR FOCUS IS TO REALLY 4931 03:16:03,720 --> 03:16:05,240 ENCOURAGE THESE YOUNG SCIENTISTS 4932 03:16:05,240 --> 03:16:08,120 TO STAY IN PARKINSON'S DISEASE, 4933 03:16:08,120 --> 03:16:10,520 BE SUCCESSFUL AT IT. 4934 03:16:10,520 --> 03:16:12,960 TO BE REALLY THE AMBASSADOR FOR 4935 03:16:12,960 --> 03:16:15,120 OUR FOUNDATION. 4936 03:16:15,120 --> 03:16:19,680 IF YOU LOOK AT OUR POSTDOCTORAL 4937 03:16:19,680 --> 03:16:21,200 FELLOWS, WE'VE BEEN GIVING SINCE 4938 03:16:21,200 --> 03:16:23,720 2002, EVEN BEFORE THE FOUNDATION 4939 03:16:23,720 --> 03:16:30,640 WAS ACTUALLY A PARKINSON'S 4940 03:16:30,640 --> 03:16:33,680 FOUNDATION, OF 92 POSTDOCTORAL 4941 03:16:33,680 --> 03:16:36,920 FELLOWS FUNDED, MOST OF THEM ARE 4942 03:16:36,920 --> 03:16:38,920 STILL ACTIVE DOING PARKINSON'S 4943 03:16:38,920 --> 03:16:41,360 DISEASE RESEARCH WHICH I THINK 4944 03:16:41,360 --> 03:16:43,400 PERSONALLY IS A LOT. 4945 03:16:43,400 --> 03:16:48,920 75% OF THEM REMAIN ACTIVE IN 4946 03:16:48,920 --> 03:16:49,200 ACADEMIA. 4947 03:16:49,200 --> 03:16:55,000 WHICH WE KNOW THE CRISIS TODAY 4948 03:16:55,000 --> 03:16:55,320 IS AMAZING. 4949 03:16:55,320 --> 03:17:00,160 IF WE LOOK AT THE JUNIOR 4950 03:17:00,160 --> 03:17:06,400 P.I.s, WE'VE BEEN GIVING THIS 4951 03:17:06,400 --> 03:17:10,480 AWARD SINCE 2016, FUNDED 12, 85% 4952 03:17:10,480 --> 03:17:11,880 STILL DOING RESEARCH IN 4953 03:17:11,880 --> 03:17:14,320 PARKINSON'S DISEASE, IN CONTRAST 4954 03:17:14,320 --> 03:17:15,800 TO OTHER NEURODEGENERATIVE 4955 03:17:15,800 --> 03:17:16,080 DISEASES. 4956 03:17:16,080 --> 03:17:18,440 MORE THAN HALF HAVE ALREADY 4957 03:17:18,440 --> 03:17:23,800 ADVANCED TO ASSOCIATE PROFESSOR, 4958 03:17:23,800 --> 03:17:30,360 PROBABLY THEY HAVE GOTTEN AN 4959 03:17:30,360 --> 03:17:31,440 R01. 4960 03:17:31,440 --> 03:17:32,320 INVESTMENTS PAY OFF. 4961 03:17:32,320 --> 03:17:35,720 IF WE LOOK AT RETURN ON 4962 03:17:35,720 --> 03:17:40,880 INVESTMENT, FOR EVERY DOLLAR WE 4963 03:17:40,880 --> 03:17:49,520 HAVE SPENT IN YOUNG SCIENTISTS, 4964 03:17:49,520 --> 03:17:52,160 RETURN OF INVESTMENTS, IN 2016 4965 03:17:52,160 --> 03:17:54,120 WE BECAME THE NEW FOUNDATION, 4966 03:17:54,120 --> 03:17:56,840 RETURN ON INVESTMENT FOR EVERY 4967 03:17:56,840 --> 03:18:00,000 DOLLAR HAS BEEN 4.4. 4968 03:18:00,000 --> 03:18:01,320 OUR INVESTMENT IN SCIENCE IS 4969 03:18:01,320 --> 03:18:06,600 DRIVING FORWARD WITH MORE AND 4970 03:18:06,600 --> 03:18:09,440 MORE PARKINSON'S DISEASE 4971 03:18:09,440 --> 03:18:09,760 RESEARCH. 4972 03:18:09,760 --> 03:18:11,080 IMPORTANTLY WE GO BEYOND THE 4973 03:18:11,080 --> 03:18:13,200 GRANTS, WHY WE THINK IT'S SO 4974 03:18:13,200 --> 03:18:16,080 IMPORTANT TO INVEST IN THE 4975 03:18:16,080 --> 03:18:19,600 PEOPLE THAT WE FUND. 4976 03:18:19,600 --> 03:18:20,920 THEY FEEL LIKE THEY, YOU KNOW, 4977 03:18:20,920 --> 03:18:23,880 WE DO REALLY CARE AND WANT THEM 4978 03:18:23,880 --> 03:18:25,760 TO BE SUCCESSFUL. 4979 03:18:25,760 --> 03:18:28,680 WE ORGANIZE ANNUAL SUMMIT TO 4980 03:18:28,680 --> 03:18:31,760 TALK ABOUT THE WORK WHERE THEY 4981 03:18:31,760 --> 03:18:34,160 NETWORK, WHERE THEY NEED OUR 4982 03:18:34,160 --> 03:18:37,120 ADVISORY BOARD MEMBERS THAT THEY 4983 03:18:37,120 --> 03:18:38,080 BECOME MENTORS FOR THEM. 4984 03:18:38,080 --> 03:18:41,120 THIS IS THESE ARE PICTURES OF 4985 03:18:41,120 --> 03:18:41,680 THEM DURING COVID. 4986 03:18:41,680 --> 03:18:45,240 IT WAS TOWARDS THE END OF THE 4987 03:18:45,240 --> 03:18:48,280 SECOND YEAR, SHOWING SCIENTIFIC 4988 03:18:48,280 --> 03:18:49,400 WORK TO SOME OF ADVISORY BOARD 4989 03:18:49,400 --> 03:18:52,120 MEMBERS AND PEOPLE FROM THE 4990 03:18:52,120 --> 03:18:55,320 FOUNDATIONS. 4991 03:18:55,320 --> 03:18:59,160 THIS IS A COHORT. 4992 03:18:59,160 --> 03:19:01,760 WE SUBSIDIZE ATTENDANCE TO 4993 03:19:01,760 --> 03:19:04,200 SCIENTIFIC CONFERENCES. 4994 03:19:04,200 --> 03:19:06,160 THESE ARE SOCIETY FOR 4995 03:19:06,160 --> 03:19:12,200 NEUROSCIENCE MEETING LAST YEAR, 4996 03:19:12,200 --> 03:19:15,440 SOME DOING POSTERS, POSTDOCTORAL 4997 03:19:15,440 --> 03:19:17,680 FELLOWSHIP AWARDEES, SUMMER 4998 03:19:17,680 --> 03:19:19,960 AWARDEES, GRATEFUL BECAUSE WE 4999 03:19:19,960 --> 03:19:23,280 PAID ATTENDANCE TO CONFERENCE. 5000 03:19:23,280 --> 03:19:26,120 WE ALSO AMPLIFIED SUCCESS IN 5001 03:19:26,120 --> 03:19:27,000 SOCIAL MEDIA. 5002 03:19:27,000 --> 03:19:29,320 IF THEY GET AN AWARD WE SAY 5003 03:19:29,320 --> 03:19:31,040 CONGRATULATIONS AND WHY. 5004 03:19:31,040 --> 03:19:34,240 IF WE WANT TO SAY THEY PUBLISHED 5005 03:19:34,240 --> 03:19:36,080 SOMETHING, WE WANT TO READ THEIR 5006 03:19:36,080 --> 03:19:39,920 PAPERS, WE DO IT. 5007 03:19:39,920 --> 03:19:44,840 IF THEY ARE A CONFERENCE, CITED 5008 03:19:44,840 --> 03:19:46,280 FOR NEUROSCIENCE, IF THEY WROTE 5009 03:19:46,280 --> 03:19:48,560 A REVIEW WE PUT THAT IN OUR 5010 03:19:48,560 --> 03:19:49,040 TWITTER. 5011 03:19:49,040 --> 03:19:52,280 AND WE ALSO PROVIDE PROFESSIONAL 5012 03:19:52,280 --> 03:19:54,680 DEVELOPMENT OPPORTUNITIES TO 5013 03:19:54,680 --> 03:19:54,880 THEM. 5014 03:19:54,880 --> 03:19:59,720 EITHER AT OUR SUMMIT OR THROUGH 5015 03:19:59,720 --> 03:20:01,360 WEBINARS OR STUFF. 5016 03:20:01,360 --> 03:20:03,880 WE ALLOW POSTDOCS TO BE 5017 03:20:03,880 --> 03:20:05,160 REVIEWERS IN OUR PANELS. 5018 03:20:05,160 --> 03:20:07,360 WE THINK IT'S A GREAT 5019 03:20:07,360 --> 03:20:09,800 OPPORTUNITY FOR THEM. 5020 03:20:09,800 --> 03:20:12,400 AND WE HAVE PROVIDED OTHER TYPES 5021 03:20:12,400 --> 03:20:13,720 OF WORKSHOPS THAT WE THINK WILL 5022 03:20:13,720 --> 03:20:17,120 ALLOW THEM TO BE SUCCESSFUL IN 5023 03:20:17,120 --> 03:20:21,160 THEIR SCIENTIFIC COMMUNITIES. 5024 03:20:21,160 --> 03:20:22,800 YOU CAN COME AND TALK TO ME 5025 03:20:22,800 --> 03:20:25,640 LATER OR IF YOU HAVE ANY 5026 03:20:25,640 --> 03:20:28,080 PARTICULAR QUESTIONS ABOUT OUR 5027 03:20:28,080 --> 03:20:33,760 GRANTS OR FELLOWSHIP FOUNDATION, 5028 03:20:33,760 --> 03:20:36,720 YOU CAN E-MAIL US. 5029 03:20:36,720 --> 03:20:42,760 5030 03:20:42,760 --> 03:20:45,920 5031 03:20:45,920 --> 03:20:54,360 >> PAUL GROSS WITH IT'S CEREBRAL 5032 03:20:54,360 --> 03:20:55,560 PALSY RESEARCH FOUNDATION IS 5033 03:20:55,560 --> 03:20:57,080 UNABLE TO JOIN US TODAY. 5034 03:20:57,080 --> 03:21:00,040 WE'RE MISSING OUT ON HEARING THE 5035 03:21:00,040 --> 03:21:01,280 RESEARCH STRATEGIES THAT THEY 5036 03:21:01,280 --> 03:21:06,320 USE BUT WE HAVE SOME TIME FOR 5037 03:21:06,320 --> 03:21:06,640 DISCUSSION. 5038 03:21:06,640 --> 03:21:08,720 WE'RE OPENING THE FLOOR, ANY 5039 03:21:08,720 --> 03:21:10,360 QUESTIONS, WE'RE HAPPY TO HEAR 5040 03:21:10,360 --> 03:21:13,520 FROM YOU WHAT YOU'D LIKE THE 5041 03:21:13,520 --> 03:21:14,840 PANEL TO DISCUSS. 5042 03:21:14,840 --> 03:21:20,200 I'M ALWAYS HAPPY TO TALK EVEN 5043 03:21:20,200 --> 03:21:22,040 IF -- I'LL KEEP ASKING QUESTIONS 5044 03:21:22,040 --> 03:21:23,480 UNLESS SOMEBODY STARTS ASKING 5045 03:21:23,480 --> 03:21:23,680 THEM. 5046 03:21:23,680 --> 03:21:26,760 I HAVE A VERY QUESTION FOR 5047 03:21:26,760 --> 03:21:28,200 CHRISTINE, DIVE INTO THE 5048 03:21:28,200 --> 03:21:30,600 CATEGORIES OF WHAT'S IN NIH 5049 03:21:30,600 --> 03:21:31,120 REPORTER. 5050 03:21:31,120 --> 03:21:40,280 I ASK THAT FROM PAST EXPERIENCE 5051 03:21:40,280 --> 03:21:42,000 WHERE EPILEPSY, FOR EXAMPLE, 5052 03:21:42,000 --> 03:21:43,200 WHICH TYPES ARE BEING FUNDED BUT 5053 03:21:43,200 --> 03:21:47,160 IN TERMS OF WHICH TYPES OF 5054 03:21:47,160 --> 03:21:50,000 RESEARCH WITHIN EPILEPSY ARE 5055 03:21:50,000 --> 03:21:50,680 BEING REFOUNDED. 5056 03:21:50,680 --> 03:21:54,280 IT'S INTERESTING TO BE ABLE TO 5057 03:21:54,280 --> 03:21:55,680 UNDERSTAND WHERE BASIC SCIENCE 5058 03:21:55,680 --> 03:21:58,000 FUNDING IS VERSUS MAYBE OUTCOMES 5059 03:21:58,000 --> 03:21:59,840 RESEARCH TYPES OF FUNDING VERSUS 5060 03:21:59,840 --> 03:22:02,360 SO MANY TYPES OF RESEARCH 5061 03:22:02,360 --> 03:22:03,240 FUNDING, EPIDEMIOLOGY. 5062 03:22:03,240 --> 03:22:05,000 CAN YOU TALK ABOUT THE OTHER 5063 03:22:05,000 --> 03:22:12,560 CATEGORIES OF WHAT'S IN NIH 5064 03:22:12,560 --> 03:22:12,840 REPORTER? 5065 03:22:12,840 --> 03:22:14,880 NIH REPORTER, IT'S ALL OF THE 5066 03:22:14,880 --> 03:22:15,720 WANTS WE FUND. 5067 03:22:15,720 --> 03:22:17,720 IT'S A TOOL THAT IS A DATABASE 5068 03:22:17,720 --> 03:22:21,840 FOR ALL THE GRANTS THAT ARE 5069 03:22:21,840 --> 03:22:22,360 FUNDED. 5070 03:22:22,360 --> 03:22:24,920 THERE ARE SORT OF PROJECT TERMS 5071 03:22:24,920 --> 03:22:26,800 THAT CAN BE AUTOMATICALLY 5072 03:22:26,800 --> 03:22:30,280 APPLIED THAT ARE AUTOMATICALLY 5073 03:22:30,280 --> 03:22:31,800 APPLIED THROUGH AN ALGORITHM TO 5074 03:22:31,800 --> 03:22:33,920 THE GRANTS SO YOU CAN SEARCH ON 5075 03:22:33,920 --> 03:22:35,760 MULTIPLE PROJECT TERMS IF YOU 5076 03:22:35,760 --> 03:22:39,360 WANT TO, SAY, IF YOU'RE LOOKING 5077 03:22:39,360 --> 03:22:42,760 AT FTD AND WANT TO UNDERSTAND IF 5078 03:22:42,760 --> 03:22:47,040 THERE ARE CLINICAL RESEARCH, I 5079 03:22:47,040 --> 03:22:51,200 WOULD SAY FOR MOST OF THESE 5080 03:22:51,200 --> 03:22:54,600 THINGS YOU STILL NEED TO DO SOME 5081 03:22:54,600 --> 03:22:57,640 MANUAL CURATION TO MAKE SURE IT 5082 03:22:57,640 --> 03:23:00,200 MEANS WHAT YOU THINK IT MEANS. 5083 03:23:00,200 --> 03:23:03,120 I THINK REPORTER AND ALL THESE 5084 03:23:03,120 --> 03:23:05,080 SEARCHES AND SEARCHING TOOLS ARE 5085 03:23:05,080 --> 03:23:07,280 GOOD TO GET YOU A FIRST 5086 03:23:07,280 --> 03:23:08,600 APPROXIMATION OF WHAT'S THERE. 5087 03:23:08,600 --> 03:23:11,480 IF YOU WANT TO GO MORE DEEPLY 5088 03:23:11,480 --> 03:23:13,080 YOU HAVE TO DOWNLOAD MANICALLY 5089 03:23:13,080 --> 03:23:15,560 AND DO IT. 5090 03:23:15,560 --> 03:23:17,280 WE DON'T DO IS THAT LEVEL FOR 5091 03:23:17,280 --> 03:23:21,200 ALL OF THE GRANTS, FOR ALL OF 5092 03:23:21,200 --> 03:23:23,920 THE AREAS, AND PART IS SO I DO A 5093 03:23:23,920 --> 03:23:26,920 LOT OF THIS KIND OF TRYING TO 5094 03:23:26,920 --> 03:23:27,880 UNDERSTAND PORTFOLIO THINGS, AND 5095 03:23:27,880 --> 03:23:31,080 EVERYBODY HAS SORT OF A 5096 03:23:31,080 --> 03:23:33,400 DIFFERENT NEED AND DIFFERENT -- 5097 03:23:33,400 --> 03:23:34,600 IT'S REALLY SPECIFIC TO THE 5098 03:23:34,600 --> 03:23:35,640 QUESTION YOU'RE TRYING TO ANSWER 5099 03:23:35,640 --> 03:23:45,440 IN A LOT OF THESE CASES. 5100 03:23:45,440 --> 03:23:53,520 I WOULD -- SEARCH TERMS ARE 5101 03:23:53,520 --> 03:23:53,800 POWERFUL. 5102 03:23:53,800 --> 03:23:54,920 LOOK CLOSELY AND THINK WHAT ARE 5103 03:23:54,920 --> 03:23:58,240 THE QUESTIONS YOU'RE TRYING TO 5104 03:23:58,240 --> 03:23:58,760 ANSWER. 5105 03:23:58,760 --> 03:23:59,760 THANK YOU. 5106 03:23:59,760 --> 03:24:02,600 WE HAVE A COUPLE QUESTIONS FROM 5107 03:24:02,600 --> 03:24:05,200 THE AUDIENCE. 5108 03:24:05,200 --> 03:24:12,640 5109 03:24:12,640 --> 03:24:17,040 >> MICHAEL PERLMUTTER, CEREBRAL 5110 03:24:17,040 --> 03:24:18,680 PALSY, A CO-WORKER IS SORRY HE 5111 03:24:18,680 --> 03:24:20,680 COULD NOT MAKE IT TODAY. 5112 03:24:20,680 --> 03:24:23,080 MY QUESTION IS SPECIFICALLY FOR 5113 03:24:23,080 --> 03:24:23,920 YOU. 5114 03:24:23,920 --> 03:24:28,120 HOW DO YOU IDENTIFY THE 5115 03:24:28,120 --> 03:24:29,080 ORGANIZATIONS YOU LAUNCH 5116 03:24:29,080 --> 03:24:30,520 PARTNERSHIPS WITH AND WHAT DOES 5117 03:24:30,520 --> 03:24:34,880 THE PROCESS LOOK LIKE? 5118 03:24:34,880 --> 03:24:35,960 IT'S SOMETIMES AWKWARD TO 5119 03:24:35,960 --> 03:24:38,040 APPROACH AN ORGANIZATION THAT IS 5120 03:24:38,040 --> 03:24:46,680 MAYBE PERCEIVED AS COMPETING FOR 5121 03:24:46,680 --> 03:24:48,320 FUNDING WITH YOU. 5122 03:24:48,320 --> 03:24:49,840 >> THANK YOU FOR THE QUESTION. 5123 03:24:49,840 --> 03:24:50,520 WE'VE ESTABLISHED PARTNERSHIPS 5124 03:24:50,520 --> 03:24:54,480 IN A NUMBER OF WAYS, AND THE 5125 03:24:54,480 --> 03:24:56,520 PARTNERSHIP WITH ADDF PREDATES 5126 03:24:56,520 --> 03:25:00,720 ME, SO THAT WAS AN INTERACTION, 5127 03:25:00,720 --> 03:25:01,600 A PERSONAL CONNECTION BETWEEN 5128 03:25:01,600 --> 03:25:04,560 FOLKS IN OUR TWO ORGANIZATIONS. 5129 03:25:04,560 --> 03:25:11,760 IN THE CASE OF THE DIGITAL TOOLS 5130 03:25:11,760 --> 03:25:12,960 PARTNERSHIP WE WANTED TO DO A 5131 03:25:12,960 --> 03:25:14,800 PARTNERSHIP IN THE ALS FIELD, 5132 03:25:14,800 --> 03:25:18,320 PARTICULARLY INTERESTED IN BEING 5133 03:25:18,320 --> 03:25:20,960 ABLE TO FIND ASSESSMENTS THAT 5134 03:25:20,960 --> 03:25:22,720 WOULD SPAN THAT WHOLE ENTIRE 5135 03:25:22,720 --> 03:25:23,000 SPECTRUM. 5136 03:25:23,000 --> 03:25:26,000 SO WE WERE ABLE TO REACH OUT TO 5137 03:25:26,000 --> 03:25:27,960 THE ALS ASSOCIATION AND THEY 5138 03:25:27,960 --> 03:25:30,600 WERE EAGER TO JOIN IN WITH US. 5139 03:25:30,600 --> 03:25:33,320 SO SOMETIMES IT COMES FROM 5140 03:25:33,320 --> 03:25:35,400 WANTING TO MEET A SPECIFIC GOAL 5141 03:25:35,400 --> 03:25:39,760 IN REACHING OUT TO A POTENTIAL 5142 03:25:39,760 --> 03:25:40,840 PARTNER ORGANIZATION. 5143 03:25:40,840 --> 03:25:51,360 SOMETIMES THEY APPROACH US AND 5144 03:25:51,600 --> 03:25:52,040 EVOLVE ORGANICALLY. 5145 03:25:52,040 --> 03:25:55,560 >> WE HELD A MEETING ON DIGITAL 5146 03:25:55,560 --> 03:25:59,280 TOOLS, THE FTD WAS CREATING 5147 03:25:59,280 --> 03:26:00,360 THEIRS, SOMETIMES HAD 5148 03:26:00,360 --> 03:26:01,080 OVERLAPPING PATIENT POPULATIONS 5149 03:26:01,080 --> 03:26:03,760 SO AROSE FROM THAT AND THE ALS 5150 03:26:03,760 --> 03:26:04,960 ASSOCIATION WAS INVITED TO THAT 5151 03:26:04,960 --> 03:26:07,040 SO WHEN WE ORGANIZE THE OUR 5152 03:26:07,040 --> 03:26:09,760 MEETINGS INVITED THOSE FOLKS TO 5153 03:26:09,760 --> 03:26:09,960 COME. 5154 03:26:09,960 --> 03:26:17,640 AND BE PART OF THE LEARNINGS AND 5155 03:26:17,640 --> 03:26:17,960 DISCUSSION. 5156 03:26:17,960 --> 03:26:18,880 >> AMERICAN EPILEPSY FOUNDATION. 5157 03:26:18,880 --> 03:26:21,600 CURIOUS ON THE ROLE OF PEOPLE 5158 03:26:21,600 --> 03:26:23,120 WITH LIVED EXPERIENCE IN THE 5159 03:26:23,120 --> 03:26:25,960 SETTING OF YOUR RESEARCH AGENDA 5160 03:26:25,960 --> 03:26:29,040 OR IN THE REVIEW OF YOUR 5161 03:26:29,040 --> 03:26:32,080 RESEARCH GRANTS OR SELECTION OF 5162 03:26:32,080 --> 03:26:33,880 THOSE RESEARCHERS ENGAGED IN ANY 5163 03:26:33,880 --> 03:26:37,440 OF OUR FELLOWSHIP PROGRAMS. 5164 03:26:37,440 --> 03:26:43,440 5165 03:26:43,440 --> 03:26:44,680 5166 03:26:44,680 --> 03:26:48,360 >> YES, IN OUR REVIEW PANELS, WE 5167 03:26:48,360 --> 03:26:53,000 DO INCLUDE IN ALL OF THEM 5168 03:26:53,000 --> 03:26:53,840 PATIENT ADVOCATES. 5169 03:26:53,840 --> 03:26:58,680 WE TRY TO HAVE AT LEAST ONE, 5170 03:26:58,680 --> 03:26:59,760 PREFERABLY TWO. 5171 03:26:59,760 --> 03:27:03,720 THE PARKINSON'S FOUNDATION HAS A 5172 03:27:03,720 --> 03:27:06,080 WHOLE SEPARATE DEPARTMENT THAT 5173 03:27:06,080 --> 03:27:11,800 RECRUITS UNTRAINED PATIENT 5174 03:27:11,800 --> 03:27:13,320 ADVOCATES YOU WHO TO PARTICIPATE 5175 03:27:13,320 --> 03:27:14,880 IN THE REVIEW PROCESS, MEET WITH 5176 03:27:14,880 --> 03:27:17,800 THEM ONCE OR TWICE BEFORE REVIEW 5177 03:27:17,800 --> 03:27:23,720 PANELS, MAKE SURE THEY REMEMBER 5178 03:27:23,720 --> 03:27:25,920 HOW TO ACCESS SUBMISSION 5179 03:27:25,920 --> 03:27:26,200 PLATFORM. 5180 03:27:26,200 --> 03:27:29,040 AND I USUALLY MEET WITH THEM TO 5181 03:27:29,040 --> 03:27:31,920 MAKE SURE THAT THEY HAVE THE 5182 03:27:31,920 --> 03:27:33,240 MATERIALS, THAT THEY READ 5183 03:27:33,240 --> 03:27:34,320 THROUGH PROPOSALS BUT DO INVOLVE 5184 03:27:34,320 --> 03:27:37,640 THEM IN ALL OF OUR REVIEW 5185 03:27:37,640 --> 03:27:37,960 PANELS. 5186 03:27:37,960 --> 03:27:42,520 WE TRY FOR THEM TO REVIEW AS 5187 03:27:42,520 --> 03:27:43,880 MANY OF THE PROPOSALS THAT THEY 5188 03:27:43,880 --> 03:27:45,160 CAN IN THE PANEL. 5189 03:27:45,160 --> 03:27:51,440 THEY USUALLY DON'T GET TO REVIEW 5190 03:27:51,440 --> 03:27:52,280 ALL OF THEM. 5191 03:27:52,280 --> 03:27:56,120 BUT THEY DO HAVE AN ACTIVE ROLE. 5192 03:27:56,120 --> 03:27:59,080 AND EVEN IF THEY HAVEN'T READ 5193 03:27:59,080 --> 03:28:01,800 ALL THE PROPOSALS, WE MAKE SURE 5194 03:28:01,800 --> 03:28:03,560 THAT IN ALL THE REVIEW PANELS 5195 03:28:03,560 --> 03:28:06,720 WHEN THEY HEAR THE DISCUSSION 5196 03:28:06,720 --> 03:28:13,080 THEY ARE ASKED ABOUT WHAT THEY 5197 03:28:13,080 --> 03:28:13,280 THINK. 5198 03:28:13,280 --> 03:28:15,280 AND SOMETIMES, YOU KNOW, WHEN 5199 03:28:15,280 --> 03:28:16,920 REVIEWERS ARE DISCUSSING 5200 03:28:16,920 --> 03:28:18,760 SOMETHING AND THEY HEAR THE 5201 03:28:18,760 --> 03:28:20,520 PATIENT ADVOCATE, EITHER A 5202 03:28:20,520 --> 03:28:23,600 PERSON WITH PARKINSON'S OR 5203 03:28:23,600 --> 03:28:25,400 CAREGIVER, IT REALLY CHANGES, 5204 03:28:25,400 --> 03:28:28,880 YOU KNOW, HOW THEY SEE A 5205 03:28:28,880 --> 03:28:29,880 PROPOSAL WHEN THEY THINK, YOU 5206 03:28:29,880 --> 03:28:32,880 KNOW, IT'S NOT REALLY RELEVANT 5207 03:28:32,880 --> 03:28:34,360 OR THEY HAD A LOT MORE TO SAY 5208 03:28:34,360 --> 03:28:40,040 THAN THEY THOUGHT THEY WOULD. 5209 03:28:40,040 --> 03:28:41,120 >> MANISH, DO YOU WANT TO ANSWER 5210 03:28:41,120 --> 03:28:42,040 THE QUESTION ONLINE? 5211 03:28:42,040 --> 03:28:42,880 DID YOU HEAR IT? 5212 03:28:42,880 --> 03:28:44,480 >> CAN YOU REPEAT IT PLEASE? 5213 03:28:44,480 --> 03:28:46,000 I COULDN'T QUITE HEAR THAT. 5214 03:28:46,000 --> 03:28:47,640 >> THE QUESTION WAS ABOUT THE 5215 03:28:47,640 --> 03:28:50,160 INCLUSION OF PEOPLE WITH LIVED 5216 03:28:50,160 --> 03:28:51,720 EXPERIENCE IN THE DECISIONS 5217 03:28:51,720 --> 03:28:53,800 AROUND WHAT IS THE OVERARCHING 5218 03:28:53,800 --> 03:28:55,000 GRANT PORTFOLIO AS WELL AS 5219 03:28:55,000 --> 03:29:01,440 DECISIONS ON SPECIFIC GRANTS. 5220 03:29:01,440 --> 03:29:02,360 5221 03:29:02,360 --> 03:29:04,520 >> WE'RE A RESEARCH FOCUSED 5222 03:29:04,520 --> 03:29:04,960 ORGANIZATION. 5223 03:29:04,960 --> 03:29:06,720 IN OUR PARADIGM, AS I'VE 5224 03:29:06,720 --> 03:29:08,240 MENTIONED IN THE PRESENTATION, 5225 03:29:08,240 --> 03:29:10,080 WE HAVE THE INDEPENDENT REVIEW 5226 03:29:10,080 --> 03:29:14,800 COMMITTEE AND WE HAVE SCIENTISTS 5227 03:29:14,800 --> 03:29:15,320 THERE. 5228 03:29:15,320 --> 03:29:16,880 SO WE HAVE TAKEN INTEREST FROM 5229 03:29:16,880 --> 03:29:18,440 PEOPLE WITH LIVED EXPERIENCE 5230 03:29:18,440 --> 03:29:23,240 WHEN IT COMES TO CORE 5231 03:29:23,240 --> 03:29:24,560 FACILITIES, AND IN 5232 03:29:24,560 --> 03:29:25,440 COMMUNICATIONS, IN TAKING 5233 03:29:25,440 --> 03:29:26,880 DECISIONS ON GRANTS THAT IS AN 5234 03:29:26,880 --> 03:29:30,240 AREA WHERE WE HAVE RELIED 5235 03:29:30,240 --> 03:29:31,440 ENTIRELY ON PEOPLE ONE WOULD 5236 03:29:31,440 --> 03:29:33,880 CONSIDER AS EXPERTS IN THE 5237 03:29:33,880 --> 03:29:39,640 SUBJECT MATTER AT HAND. 5238 03:29:39,640 --> 03:29:39,800 5239 03:29:39,800 --> 03:29:41,400 >> THANK YOU, I'M GOING TO CHIME 5240 03:29:41,400 --> 03:29:42,720 IN AND DEFER. 5241 03:29:42,720 --> 03:29:43,840 PART OF OUR FOCUS, FOR EXAMPLE, 5242 03:29:43,840 --> 03:29:45,680 OF THE CONNECTION TO ALS IS 5243 03:29:45,680 --> 03:29:47,000 BECAUSE WE HAVE MANY PEOPLE ON 5244 03:29:47,000 --> 03:29:49,960 THE BOARD AND RESEARCH 5245 03:29:49,960 --> 03:29:51,360 COMMITTEES THAT DECIDE WHICH 5246 03:29:51,360 --> 03:29:53,600 GRANTS WE FUND, TAKE REVIEWER 5247 03:29:53,600 --> 03:29:54,960 FEEDBACK AND MAKE FINAL 5248 03:29:54,960 --> 03:29:55,240 DECISIONS. 5249 03:29:55,240 --> 03:29:59,520 A LOT OF FOLKS ARE FROM GENETIC 5250 03:29:59,520 --> 03:30:02,560 FAMILIES, DON'T HAVE MUCH LIVED 5251 03:30:02,560 --> 03:30:04,360 EXPERIENCE ABOUT HAVING EITHER 5252 03:30:04,360 --> 03:30:06,640 FTD OR ALS OR A COMBINATION 5253 03:30:06,640 --> 03:30:09,680 MANIFEST IN THEIR FAMILIES. 5254 03:30:09,680 --> 03:30:11,320 WE DON'T BRING THEM IN AS 5255 03:30:11,320 --> 03:30:12,560 REVIEWERS BUT IN DECISIONS 5256 03:30:12,560 --> 03:30:13,800 AREN'T WHERE OUR FUNDING 5257 03:30:13,800 --> 03:30:17,480 PORTFOLIO LIES. 5258 03:30:17,480 --> 03:30:19,240 >> I'LL JUST ADD IN OUR PILOT 5259 03:30:19,240 --> 03:30:21,760 GRANTS THE MOST RECENT FUNDING 5260 03:30:21,760 --> 03:30:25,680 STREAM IS A PILOT GRANT THAT 5261 03:30:25,680 --> 03:30:26,760 SPEAKS SPECIFICALLY TO 5262 03:30:26,760 --> 03:30:27,520 INTERVENTIONS FOR IMPROVING 5263 03:30:27,520 --> 03:30:29,840 QUALITY OF LIFE FOR PEOPLE WITH 5264 03:30:29,840 --> 03:30:31,920 FTD IN THEIR FAMILIES SO WE HAVE 5265 03:30:31,920 --> 03:30:34,240 A FUNDING STREAM THAT IS 5266 03:30:34,240 --> 03:30:37,760 SPECIFICALLY DIRECTED TO THEIR 5267 03:30:37,760 --> 03:30:37,960 NEEDS. 5268 03:30:37,960 --> 03:30:40,040 WE ALSO OFFER OPPORTUNITIES FOR 5269 03:30:40,040 --> 03:30:41,680 RESEARCHERS AND PEOPLE WITH 5270 03:30:41,680 --> 03:30:42,560 LIVED EXPERIENCE TO ENCOUNTER 5271 03:30:42,560 --> 03:30:44,800 ONE ANOTHER AND LEARN FROM ONE 5272 03:30:44,800 --> 03:30:45,000 ANOTHER. 5273 03:30:45,000 --> 03:30:47,800 SO, FOR EXAMPLE, WE HAVE DONE 5274 03:30:47,800 --> 03:30:49,920 FOR THE LAST SEVERAL YEARS A 5275 03:30:49,920 --> 03:30:52,440 NETWORKING EVENT AT THE SOCIETY 5276 03:30:52,440 --> 03:30:54,000 FOR NEUROSCIENCE MEETING, AND 5277 03:30:54,000 --> 03:30:55,720 THIS IS A SATELLITE EVENT. 5278 03:30:55,720 --> 03:31:00,240 SO WE ARE ABLE TO INVITE PEOPLE 5279 03:31:00,240 --> 03:31:01,440 FROM OUR COMMUNITY WHO CAN COME 5280 03:31:01,440 --> 03:31:04,080 IN AND TALK TO THE RESEARCHERS, 5281 03:31:04,080 --> 03:31:06,680 GET TO MEET THEM, VICE VERSA. 5282 03:31:06,680 --> 03:31:08,320 AND AT OUR ANNUAL EDUCATION 5283 03:31:08,320 --> 03:31:09,440 CONFERENCE THIS PAST YEAR, WHICH 5284 03:31:09,440 --> 03:31:12,360 IS A MEETING THAT IS 5285 03:31:12,360 --> 03:31:13,880 SPECIFICALLY FOR PEOPLE WITH FTD 5286 03:31:13,880 --> 03:31:15,440 AND THEIR FAMILIES, A CHANCE FOR 5287 03:31:15,440 --> 03:31:17,760 THEM TO GET MUTUAL SUPPORT, WE 5288 03:31:17,760 --> 03:31:19,840 HAD A PANEL THAT FEATURED THREE 5289 03:31:19,840 --> 03:31:21,800 OF OUR FUNDED RESEARCHERS. 5290 03:31:21,800 --> 03:31:23,680 AND THEY WERE ROCK STARS. 5291 03:31:23,680 --> 03:31:25,200 THEY DID A GREAT JOB. 5292 03:31:25,200 --> 03:31:27,840 THEY TOOK QUESTIONS FROM THE 5293 03:31:27,840 --> 03:31:30,200 AUDIENCE, AND IT WAS A WONDERFUL 5294 03:31:30,200 --> 03:31:31,680 OPPORTUNITY FOR PEOPLE TO SEE 5295 03:31:31,680 --> 03:31:33,200 THE RESEARCHERS HAVE A HUMAN 5296 03:31:33,200 --> 03:31:35,160 FACE AND FOR RESEARCHERS TO 5297 03:31:35,160 --> 03:31:37,040 UNDERSTAND WHAT FAMILIES GO 5298 03:31:37,040 --> 03:31:39,880 THROUGH. 5299 03:31:39,880 --> 03:31:42,080 >> AND I'D LIKE TO JUST ADD A 5300 03:31:42,080 --> 03:31:43,600 LITTLE BIT OF COLOR TO THE 5301 03:31:43,600 --> 03:31:45,800 QUESTION THAT PENNY AND DEBRA 5302 03:31:45,800 --> 03:31:48,120 ANSWERED ABOUT HOW YOU APPEAL TO 5303 03:31:48,120 --> 03:31:51,720 ORGANIZATIONS THAT MIGHT 5304 03:31:51,720 --> 03:31:54,360 CONSIDER YOU A COMPETITOR FOR 5305 03:31:54,360 --> 03:31:54,800 FUNDING. 5306 03:31:54,800 --> 03:31:56,640 WHEN WE FIRST ESTABLISHED FARA 5307 03:31:56,640 --> 03:31:58,040 WE KNEW THE MUSCULAR DYSTROPHY 5308 03:31:58,040 --> 03:32:00,240 ASSOCIATION WITH OUR DISEASE AS 5309 03:32:00,240 --> 03:32:10,800 ONE OF ITS 42 DISEASES. 5310 03:32:10,800 --> 03:32:11,840 WE KNEW NATIONAL ATAXIA 5311 03:32:11,840 --> 03:32:12,840 FOUNDATION WITH IT. 5312 03:32:12,840 --> 03:32:14,720 WE SAT DOWN WITH BOARDS OF 5313 03:32:14,720 --> 03:32:18,080 DIRECTORS AND TOLD THEM WE'RE 5314 03:32:18,080 --> 03:32:22,240 NOT HERE TO COMPETE, WE'RE HERE 5315 03:32:22,240 --> 03:32:26,200 TO COLLABORATE, WE CO-FUNDED 5316 03:32:26,200 --> 03:32:27,560 GRANTS, SHARED REVIEWS AND 5317 03:32:27,560 --> 03:32:28,520 PARTNERSHIPS HAVE LASTED THROUGH 5318 03:32:28,520 --> 03:32:30,440 THE 25 YEARS OF OUR EXISTENCE 5319 03:32:30,440 --> 03:32:31,640 AND WE'VE FUNDED MILLIONS OF 5320 03:32:31,640 --> 03:32:40,280 DOLLARS OF RESEARCH TOGETHER. 5321 03:32:40,280 --> 03:32:41,480 >> I'M NOT SEEING OTHER 5322 03:32:41,480 --> 03:32:41,920 QUESTIONS. 5323 03:32:41,920 --> 03:32:45,680 WE HAVE A FEW MORE MINUTES. 5324 03:32:45,680 --> 03:32:49,520 OH, WE DID HAVE ANOTHER ONE. 5325 03:32:49,520 --> 03:32:50,120 DR. KOROSHETZ? 5326 03:32:50,120 --> 03:32:51,800 >> I WAS WONDERING IF MAYBE SOME 5327 03:32:51,800 --> 03:32:54,880 OF YOU COULD TALK A LITTLE BIT 5328 03:32:54,880 --> 03:32:56,760 ABOUT KIND OF THE ORIGINS OF 5329 03:32:56,760 --> 03:32:58,400 YOUR FOUNDATION AND HOW YOU 5330 03:32:58,400 --> 03:33:02,200 PROGRESSED OVER THE YEARS. 5331 03:33:02,200 --> 03:33:05,400 CERTAINLY FTD AND PARKINSON'S 5332 03:33:05,400 --> 03:33:07,520 FOUNDATIONS HAVE BEEN AROUND FOR 5333 03:33:07,520 --> 03:33:09,120 A WHILE, BUT A LOT OF THE 5334 03:33:09,120 --> 03:33:11,520 ORGANIZATIONS HERE, SOME ARE 5335 03:33:11,520 --> 03:33:14,360 YOUNGER AND MIGHT BENEFIT FROM 5336 03:33:14,360 --> 03:33:21,080 KIND OF THE HISTORY AND WHAT 5337 03:33:21,080 --> 03:33:27,040 THINGS ALLOWED YOU TO GROW WHEE 5338 03:33:27,040 --> 03:33:27,560 YOU ARE NOW. 5339 03:33:27,560 --> 03:33:29,200 >> MANISH, DID YOU HEAR THAT? 5340 03:33:29,200 --> 03:33:29,440 >> YES. 5341 03:33:29,440 --> 03:33:31,560 >> DO YOU WANT TO TAKE THAT 5342 03:33:31,560 --> 03:33:31,760 FIRST? 5343 03:33:31,760 --> 03:33:36,360 >> SURE. 5344 03:33:36,360 --> 03:33:39,640 WE LAUNCHED IN 2013, THEY ARE 5345 03:33:39,640 --> 03:33:41,920 OFTEN LAUNCHED BY SOMEONE WHO 5346 03:33:41,920 --> 03:33:45,680 HAS BEEN PERSONALLY AFFECTED. 5347 03:33:45,680 --> 03:33:47,400 SO OUR FOUNDER THEN, THIS IS 5348 03:33:47,400 --> 03:33:49,480 PUBLIC KNOWLEDGE, LOST HIS 5349 03:33:49,480 --> 03:33:50,720 FATHER TO ALS. 5350 03:33:50,720 --> 03:33:51,800 AND THAT'S WHAT MOTIVATED HIM TO 5351 03:33:51,800 --> 03:33:56,000 DO SOMETHING ABOUT THE DISEASE 5352 03:33:56,000 --> 03:34:01,400 AND LAUNCHED THE FOUNDATION. 5353 03:34:01,400 --> 03:34:03,320 BRIEF HISTORY, BASED OUT OF 5354 03:34:03,320 --> 03:34:05,080 COLUMBIA UNIVERSITY, THE IDEA 5355 03:34:05,080 --> 03:34:06,480 FUNDS WERE FOR ANY SCIENTIST 5356 03:34:06,480 --> 03:34:08,000 AROUND THE WORLD WITH A GOOD 5357 03:34:08,000 --> 03:34:11,280 IDEA BUT WE WERE LEVERAGING 5358 03:34:11,280 --> 03:34:14,680 COLUMBIA'S INFRASTRUCTURE. 5359 03:34:14,680 --> 03:34:17,560 AROUND 2015 WE REALIZED IF WE 5360 03:34:17,560 --> 03:34:20,160 WANTED TO BE NIMBLE AND BEING 5361 03:34:20,160 --> 03:34:22,040 FAIR TO COLUMBIA UNIVERSITY, A 5362 03:34:22,040 --> 03:34:24,680 BIG UNIVERSITY INFRASTRUCTURE IS 5363 03:34:24,680 --> 03:34:25,400 NOT GEARED TOWARDS QUICK 5364 03:34:25,400 --> 03:34:26,160 DECISION MAKING. 5365 03:34:26,160 --> 03:34:29,360 SO WE SPUN OUT AND BECAME AN 5366 03:34:29,360 --> 03:34:30,680 INDEPENDENT NON-PROFIT AT THAT 5367 03:34:30,680 --> 03:34:31,360 POINT. 5368 03:34:31,360 --> 03:34:33,520 AND THEN WE HAVE BEEN ABLE TO 5369 03:34:33,520 --> 03:34:34,800 GROW GRADUALLY. 5370 03:34:34,800 --> 03:34:37,880 ONE OF THE KEYS FOR US HAS BEEN 5371 03:34:37,880 --> 03:34:39,760 WE HAVE ALWAYS TRIED TO THINK 5372 03:34:39,760 --> 03:34:43,680 DIFFERENTLY ABOUT HOW WE WANT TO 5373 03:34:43,680 --> 03:34:46,560 APPROACH THE CHALLENGES FACING 5374 03:34:46,560 --> 03:34:48,160 THE FIELD, IDENTIFYING KEY 5375 03:34:48,160 --> 03:34:50,800 BARRIERS IN OUR PRESENTATION AND 5376 03:34:50,800 --> 03:34:54,840 TRYING TO ADDRESS THEM. 5377 03:34:54,840 --> 03:34:56,480 THAT HAS ALLOWED US TO ESTABLISH 5378 03:34:56,480 --> 03:34:58,680 A POSITION OF CREDIBILITY IN THE 5379 03:34:58,680 --> 03:35:00,720 BROADER COMMUNITY, AND THAT HAS 5380 03:35:00,720 --> 03:35:04,920 ALLOWED US TO THEN GROW. 5381 03:35:04,920 --> 03:35:06,000 >> THANK YOU. 5382 03:35:06,000 --> 03:35:10,040 SPEAKING FOR THE ASSOCIATION FOR 5383 03:35:10,040 --> 03:35:10,800 FRONTOTEMPORAL DEGENERATION 5384 03:35:10,800 --> 03:35:13,440 FOUNDED BY HELEN ANN COMSTOCK, A 5385 03:35:13,440 --> 03:35:15,440 CAREGIVER FOR HER HUSBAND, 5386 03:35:15,440 --> 03:35:19,120 STARTING WITH A $1,000 DONATION, 5387 03:35:19,120 --> 03:35:19,880 15 YEARS AGO. 5388 03:35:19,880 --> 03:35:23,520 MY BOSS IS GOING TO GET MAD IF I 5389 03:35:23,520 --> 03:35:25,880 DON'T REMEMBER THE DETAILS. 5390 03:35:25,880 --> 03:35:28,800 SHE'S BEEN ACTIVE IN THE 5391 03:35:28,800 --> 03:35:31,080 ALZHEIMER'S ASSOCIATION WHICH IS 5392 03:35:31,080 --> 03:35:32,520 AN INCREDIBLE ORGANIZATION. 5393 03:35:32,520 --> 03:35:34,480 BUT WHAT SHE FELT VERY DEEPLY IS 5394 03:35:34,480 --> 03:35:37,360 THAT THE NEEDS OF PEOPLE WITH 5395 03:35:37,360 --> 03:35:39,760 FTD ARE DIFFERENT, AN EARLIER 5396 03:35:39,760 --> 03:35:43,440 ONSET DEMENTIA, SYMPTOMS CAN BE 5397 03:35:43,440 --> 03:35:43,760 DIFFERENT. 5398 03:35:43,760 --> 03:35:47,200 IF YOU'RE 50 AND DEALING WITH 5399 03:35:47,200 --> 03:35:49,240 BEHAVIORAL AND COGNITIVE -- NOT 5400 03:35:49,240 --> 03:35:50,800 MEMORY PROBLEMS BUT BEHAVIORAL 5401 03:35:50,800 --> 03:35:52,200 OR LANGUAGE OR MOTOR PROBLEMS 5402 03:35:52,200 --> 03:35:56,720 WITH SOMEBODY WHO IS DYING OF A 5403 03:35:56,720 --> 03:35:58,360 FATAL NEURODEGENERATIVE DISEASE 5404 03:35:58,360 --> 03:35:59,320 YOUR NEEDS AS CAREGIVER, OFT 5405 03:35:59,320 --> 03:36:01,640 WITHIN YOUNG KIDS AT HOME, ARE 5406 03:36:01,640 --> 03:36:02,440 DIFFERENT THAN A 70-YEAR-OLD 5407 03:36:02,440 --> 03:36:05,120 CARING FOR A HUSBAND WITH MEMORY 5408 03:36:05,120 --> 03:36:07,680 LOSS THAT'S VERY WELL UNDERSTOOD 5409 03:36:07,680 --> 03:36:11,960 BY THE NEUROLOGY COMMUNITY. 5410 03:36:11,960 --> 03:36:15,040 HELEN FOUNDED FTD WITH A 5411 03:36:15,040 --> 03:36:16,960 THOUSAND DOLLAR DONATION, A 5412 03:36:16,960 --> 03:36:19,480 LONGSTANDING VOLUNTEER, FOUND A 5413 03:36:19,480 --> 03:36:22,120 GROUP OF LIKE-MINDED FOLKS, 5414 03:36:22,120 --> 03:36:23,200 FOUND AMAZING RESEARCHERS WHO 5415 03:36:23,200 --> 03:36:25,040 UNDERSTOOD AND WERE COMMITTED TO 5416 03:36:25,040 --> 03:36:26,920 MAKING A DIFFERENCE IN THE SPACE 5417 03:36:26,920 --> 03:36:28,440 AND IT'S GROWN OVER MANY, MANY 5418 03:36:28,440 --> 03:36:28,760 YEARS. 5419 03:36:28,760 --> 03:36:30,120 WE HAVE A STAFF OF 45 PEOPLE 5420 03:36:30,120 --> 03:36:33,840 RIGHT NOW. 5421 03:36:33,840 --> 03:36:37,560 DO YOU WANT TO ANSWER ON BEHALF 5422 03:36:37,560 --> 03:36:38,600 OF PARKINSON'S FOUNDATION? 5423 03:36:38,600 --> 03:36:41,040 >> I KNOW THE FOUNDATION AS IT 5424 03:36:41,040 --> 03:36:42,720 IS NOW CALLED, PARKINSON'S 5425 03:36:42,720 --> 03:36:45,560 FOUNDATION, WAS RESULT OF A 5426 03:36:45,560 --> 03:36:47,760 MERGER FROM PARKINSON'S DISEASE 5427 03:36:47,760 --> 03:36:50,480 FOUNDATION THAT WAS IN NEW YORK 5428 03:36:50,480 --> 03:36:54,320 CITY AND I THINK IT WAS 5429 03:36:54,320 --> 03:36:54,840 INTERNATIONAL PARKINSON'S 5430 03:36:54,840 --> 03:36:56,400 FOUNDATION BASED IN MIAMI. 5431 03:36:56,400 --> 03:36:59,800 SO, I THINK THE TWO FOUNDATIONS 5432 03:36:59,800 --> 03:37:01,880 REALIZED THEY WERE BETTER 5433 03:37:01,880 --> 03:37:03,280 TOGETHER THAN BY THEMSELVES. 5434 03:37:03,280 --> 03:37:07,040 AND THEY DECIDED TO MERGE BACK 5435 03:37:07,040 --> 03:37:10,600 IN 2016, THAT'S WHEN THE NOW CEO 5436 03:37:10,600 --> 03:37:11,600 WAS HIRED. 5437 03:37:11,600 --> 03:37:17,560 HE REALLY DID A TREMENDOUS JOB 5438 03:37:17,560 --> 03:37:22,000 OF BRINGING TWO FOUNDATIONS 5439 03:37:22,000 --> 03:37:25,080 TOGETHER AND MAKING THEM INTO A 5440 03:37:25,080 --> 03:37:26,360 COHESIVE FOUNDATION THAT WORKS 5441 03:37:26,360 --> 03:37:26,960 PRIMARILY REMOTE. 5442 03:37:26,960 --> 03:37:30,000 WE WERE IN TUNE BEFORE THE 5443 03:37:30,000 --> 03:37:30,320 PANDEMIC. 5444 03:37:30,320 --> 03:37:34,240 SO, IT WAS REALLY EASY FOR US TO 5445 03:37:34,240 --> 03:37:38,440 BEAR THE PANDEMIC IN THAT WAY. 5446 03:37:38,440 --> 03:37:41,600 AND JOHN PREVIOUSLY WORKED AT 5447 03:37:41,600 --> 03:37:43,360 CYSTIC FIBROSIS FOUNDATION, THAT 5448 03:37:43,360 --> 03:37:45,760 I THINK EVERYONE KNOWS BECAUSE 5449 03:37:45,760 --> 03:37:47,200 OF THEIR INCREDIBLE SUCCESS IN 5450 03:37:47,200 --> 03:37:48,360 BRINGING DRUGS TO MARKET. 5451 03:37:48,360 --> 03:37:50,480 HE TOOK EVERYTHING HE HAD 5452 03:37:50,480 --> 03:37:52,960 LEARNED WORKING AT THE CYSTIC 5453 03:37:52,960 --> 03:37:54,160 FIBROSIS FOUNDATION, KNOWING 5454 03:37:54,160 --> 03:37:56,560 THAT PARKINSON'S FOUNDATION IS 5455 03:37:56,560 --> 03:37:59,200 NOT CYSTIC FIBROSIS, IT'S A MORE 5456 03:37:59,200 --> 03:38:01,840 COMPLEX DISEASE. 5457 03:38:01,840 --> 03:38:04,360 THE STRATEGIES WERE NOT GOING TO 5458 03:38:04,360 --> 03:38:06,120 WORK FOR PARKINSON'S BUT HE 5459 03:38:06,120 --> 03:38:09,760 MANAGED, YOU SAW THE RESULTS IN 5460 03:38:09,760 --> 03:38:13,240 THE SLIDES, A LOT OF THINGS, THE 5461 03:38:13,240 --> 03:38:19,120 FOUNDATION TOOK OFF SINCE HE 5462 03:38:19,120 --> 03:38:21,400 TOOK OVER IN 2016, IN TERMS OF 5463 03:38:21,400 --> 03:38:23,520 FINANCIALLY AND PERSONNEL. 5464 03:38:23,520 --> 03:38:25,240 WE'VE EXPANDED NOT ONLY ON THE 5465 03:38:25,240 --> 03:38:29,360 RESEARCH SIDE BUT ALSO THE 5466 03:38:29,360 --> 03:38:30,880 CLINICAL SIDE WITH CLINICAL 5467 03:38:30,880 --> 03:38:31,400 WORK. 5468 03:38:31,400 --> 03:38:36,560 ALSO ALL THE WORK WE FUND IN 5469 03:38:36,560 --> 03:38:38,640 EDUCATION AND THE CARE AND 5470 03:38:38,640 --> 03:38:43,560 THINGS WE DO FOR CAREGIVERS. 5471 03:38:43,560 --> 03:38:47,160 SO THE FOUNDATION AS IT IS NOW, 5472 03:38:47,160 --> 03:38:50,680 IT'S REALLY THE RESULT OF GREAT 5473 03:38:50,680 --> 03:38:52,520 MANAGEMENT AND THE PEOPLE THAT 5474 03:38:52,520 --> 03:38:54,520 WORK THERE. 5475 03:38:54,520 --> 03:39:09,640 RIGHT NOW IT'S AROUND 150. 5476 03:39:09,640 --> 03:39:11,160 >> I SAY WITH A RECOGNITION 5477 03:39:11,160 --> 03:39:12,960 THERE ARE OTHER COMMUNITIES 5478 03:39:12,960 --> 03:39:14,160 AHEAD OF THE HUNTINGTON'S 5479 03:39:14,160 --> 03:39:19,560 DISEASE COMMUNITY, WHEN IT COMES 5480 03:39:19,560 --> 03:39:21,960 TO HAVING DISEASE-MODIFYING 5481 03:39:21,960 --> 03:39:23,160 THERAPIES, OR LEVELS OF 5482 03:39:23,160 --> 03:39:27,280 TRANSFORMATIVE CARE TOOLS FOR 5483 03:39:27,280 --> 03:39:28,200 THEIR PATIENT POPULATIONS. 5484 03:39:28,200 --> 03:39:31,440 TODAY YOU'RE GOING TO HEAR FROM 5485 03:39:31,440 --> 03:39:35,080 MELISSA KENNEDY, TALKING ABOUT A 5486 03:39:35,080 --> 03:39:35,720 BREAKTHROUGH IN TREATMENT OF 5487 03:39:35,720 --> 03:39:37,840 RETT SYNDROME. 5488 03:39:37,840 --> 03:39:40,240 YOU'LL HEAR FROM RON BARTEK 5489 03:39:40,240 --> 03:39:41,720 TALKING ABOUT THE RECENT 5490 03:39:41,720 --> 03:39:44,360 APPROVAL OF A DRUG FOR 5491 03:39:44,360 --> 03:39:45,680 FRIEDRICH'S ATAXIA. 5492 03:39:45,680 --> 03:39:48,320 AND TWO SPEAKERS WHO FOR 5493 03:39:48,320 --> 03:39:53,920 DIFFERENT REASONS ARE UNABLE TO 5494 03:39:53,920 --> 03:39:59,160 BE HERE BUT NEIL THAKUR TALKING 5495 03:39:59,160 --> 03:40:00,880 ABOUT STRATEGIES FOR ALS AND 5496 03:40:00,880 --> 03:40:03,960 FOLLOWED BY DR. TIM MILLER FROM 5497 03:40:03,960 --> 03:40:05,920 WASHINGTON UNIVERSITY, WHO HAS A 5498 03:40:05,920 --> 03:40:06,880 RECORDED PRESENTATION BUT IS 5499 03:40:06,880 --> 03:40:17,320 GOING TO JOIN LIVE FOR THE 5500 03:40:20,120 --> 03:40:20,360 CONVERSATION. 5501 03:40:20,360 --> 03:40:27,360 EACH HAS TAKEN ADVANTAGE OF 5502 03:40:27,360 --> 03:40:28,040 ACCELERATED APPROVAL. 5503 03:40:28,040 --> 03:40:30,640 THERE'S A LOT OF MAGIC AND LUCK 5504 03:40:30,640 --> 03:40:32,600 AND DETERMINATION IN THE DRUG 5505 03:40:32,600 --> 03:40:32,800 PROCESS. 5506 03:40:32,800 --> 03:40:35,360 WE DON'T HAVE CORPORATE 5507 03:40:35,360 --> 03:40:37,160 REPRESENTATIVES IN THE ROOM, 5508 03:40:37,160 --> 03:40:38,640 ANOTHER PART OF THE PARTNERSHIP 5509 03:40:38,640 --> 03:40:43,480 THAT MAKES THIS POSSIBLE. 5510 03:40:43,480 --> 03:40:45,200 BUT FROM THE IDENTIFICATION OF 5511 03:40:45,200 --> 03:40:46,680 POTENTIAL STRATEGY FOR DRUG 5512 03:40:46,680 --> 03:40:48,800 DEVELOPMENT TO THE PRE-CLINICAL 5513 03:40:48,800 --> 03:40:52,200 WORK, MOUSE MODELS, TO THE 5514 03:40:52,200 --> 03:40:54,520 SAFETY AND EFFICACY TRIALS, 5515 03:40:54,520 --> 03:40:55,840 PHASE 1, 2, 3, AND THEN IF 5516 03:40:55,840 --> 03:40:58,360 YOU'RE ABLE TO GET TO 4 THROUGH 5517 03:40:58,360 --> 03:41:00,000 THE POST MARKETING APPROVAL IT'S 5518 03:41:00,000 --> 03:41:03,920 A REALLY LONG PROCESS. 5519 03:41:03,920 --> 03:41:07,440 WE ALL KNOW THAT. 5520 03:41:07,440 --> 03:41:08,760 ACCELERATED APPROVAL CAN PROVIDE 5521 03:41:08,760 --> 03:41:10,480 NOT A SHORTCUT BUT MAYBE A FAST 5522 03:41:10,480 --> 03:41:13,320 TRACK FOR SPECIAL PROGRAMS ABLE 5523 03:41:13,320 --> 03:41:15,520 TO SHOW ABILITY TO INFLUENCE 5524 03:41:15,520 --> 03:41:16,960 DISEASE PROGRESSION AND HAVE 5525 03:41:16,960 --> 03:41:21,520 MEANINGFUL IMPACT ON OUR 5526 03:41:21,520 --> 03:41:22,040 FAMILIES. 5527 03:41:22,040 --> 03:41:22,240 EARLY. 5528 03:41:22,240 --> 03:41:23,320 WE LEARN FROM THIS. 5529 03:41:23,320 --> 03:41:25,160 THAT'S WHAT TODAY'S SESSION IS 5530 03:41:25,160 --> 03:41:27,160 ALL ABOUT. 5531 03:41:27,160 --> 03:41:29,360 WE'RE GOING TILL 4:00, 4:10, TO 5532 03:41:29,360 --> 03:41:30,640 MAKE SURE WE HAVE TIME FOR 5533 03:41:30,640 --> 03:41:38,960 WALTER TO TAKE US HOME TODAY. 5534 03:41:38,960 --> 03:41:40,520 EACH SPEAKER WILL SHOWCASE 5535 03:41:40,520 --> 03:41:43,120 STORIES IN DRUG APPROVAL AND 5536 03:41:43,120 --> 03:41:44,120 WE'LL HAVE GROUP CONVERSATION. 5537 03:41:44,120 --> 03:41:46,000 I'LL TURN IT OVER TO MELISSA TO 5538 03:41:46,000 --> 03:41:46,960 GET US GOING. 5539 03:41:46,960 --> 03:41:55,320 THANKS, EVERYONE. 5540 03:41:55,320 --> 03:41:59,360 5541 03:41:59,360 --> 03:42:03,440 >> IT'S AN HONOR TO BE HERE 5542 03:42:03,440 --> 03:42:03,640 TODAY. 5543 03:42:03,640 --> 03:42:08,920 WHICH MIC IS BEST? 5544 03:42:08,920 --> 03:42:10,320 BOTH OF THEM, OKAY. 5545 03:42:10,320 --> 03:42:15,600 SEVERAL COMMENTS HAVE BEEN MADE. 5546 03:42:15,600 --> 03:42:18,240 I HAVE THE DISTINCT HONOR OF 5547 03:42:18,240 --> 03:42:21,720 SERVING EVERY DAY AS THE CEO OF 5548 03:42:21,720 --> 03:42:22,600 INTERNATIONAL RETT SYNDROME 5549 03:42:22,600 --> 03:42:23,000 FOUNDATION. 5550 03:42:23,000 --> 03:42:26,280 I'VE ONLY BEEN IN THIS ROLE FOR 5551 03:42:26,280 --> 03:42:26,560 SIX YEARS. 5552 03:42:26,560 --> 03:42:31,480 I SAY "ONLY" BECAUSE THE DRUG 5553 03:42:31,480 --> 03:42:33,200 DEVELOPMENT PROCESS EVEN 5554 03:42:33,200 --> 03:42:34,880 ACCELERATED IS EXCRUCIATINGLY 5555 03:42:34,880 --> 03:42:35,840 LONG AND PAINFUL. 5556 03:42:35,840 --> 03:42:37,800 I'M ONLY HERE RIGHT NOW BECAUSE 5557 03:42:37,800 --> 03:42:40,640 OF THE DETERMINATION AND HARD 5558 03:42:40,640 --> 03:42:46,680 WORK OF BOARD MEMBERS OF IRSF 5559 03:42:46,680 --> 03:42:48,080 MAKING CRITICAL DECISIONS IN 5560 03:42:48,080 --> 03:42:48,600 2010. 5561 03:42:48,600 --> 03:42:51,840 I'M ONLY HERE BECAUSE OF THE 5562 03:42:51,840 --> 03:43:02,120 VERY BRAVE PATIENT COMMUNITY AND 5563 03:43:02,120 --> 03:43:04,400 CAREGIVER COMMUNITY THAT 5564 03:43:04,400 --> 03:43:06,160 PARTICIPATED IN CLINICAL TRIALS, 5565 03:43:06,160 --> 03:43:08,000 I FEEL LIKE AN IMPOSTOR WHO CAME 5566 03:43:08,000 --> 03:43:10,360 IN AT THE END OF A GREAT STORY. 5567 03:43:10,360 --> 03:43:13,320 LIKE MANY RARE DISEASE 5568 03:43:13,320 --> 03:43:16,160 FOUNDATIONS, OURS HAD VERY 5569 03:43:16,160 --> 03:43:19,840 HUMBLE BEGINNINGS 40 YEARS AGO, 5570 03:43:19,840 --> 03:43:21,600 198 3, KITCHEN TABLE OF KATHY 5571 03:43:21,600 --> 03:43:25,440 HUNTER, A MOM OF A BEAUTIFUL 5572 03:43:25,440 --> 03:43:26,840 YOUNG GIRL DIAGNOSED WITH RETT 5573 03:43:26,840 --> 03:43:30,120 SYNDROME, THE KITCHEN TABLE IS 5574 03:43:30,120 --> 03:43:31,240 NOT FAR FROM HERE, SO IT'S AN 5575 03:43:31,240 --> 03:43:32,760 HONOR TO BE HERE WITH YOU. 5576 03:43:32,760 --> 03:43:34,520 OVER THE 40-YEAR PERIOD OF TIME 5577 03:43:34,520 --> 03:43:36,720 OUR BOARD HAS GROWN AND MATURED. 5578 03:43:36,720 --> 03:43:42,080 WE NOW HAVE A BOARD THAT IS MADE 5579 03:43:42,080 --> 03:43:46,800 OF CAREGIVERS, PARENTS, AND 5580 03:43:46,800 --> 03:43:47,560 AUNTS, UNCLES, SCIENTISTS, 5581 03:43:47,560 --> 03:43:53,840 CLINICIANS, ALSO RECENTLY 5582 03:43:53,840 --> 03:43:54,480 BIOTECH INVESTORS, HELPFUL IN 5583 03:43:54,480 --> 03:43:55,360 THE NEW PHASE. 5584 03:43:55,360 --> 03:44:01,360 WE HAVE A DUAL MISSION OF 5585 03:44:01,360 --> 03:44:03,200 ACCELERATING MEANINGFUL AND 5586 03:44:03,200 --> 03:44:05,080 POTENTIALLY HELPFUL RESEARCH, 5587 03:44:05,080 --> 03:44:06,280 WHILE SUPPORTING, EDUCATING, 5588 03:44:06,280 --> 03:44:09,640 EMPOWERING AND ADVOCATING FOR 5589 03:44:09,640 --> 03:44:11,160 FAMILIES. 5590 03:44:11,160 --> 03:44:12,480 OUR RESEARCH IS STRAIGHTFORWARD, 5591 03:44:12,480 --> 03:44:19,760 MULTIPLE SHOTS ON GOAL, AND NO 5592 03:44:19,760 --> 03:44:21,920 ONE GETS LEFT BEHIND. 5593 03:44:21,920 --> 03:44:23,280 WE HAVE THREE PRONGS TO THE 5594 03:44:23,280 --> 03:44:24,120 RESEARCH STRATEGY. 5595 03:44:24,120 --> 03:44:26,760 THIS IS REALLY IMPORTANT BECAUSE 5596 03:44:26,760 --> 03:44:29,120 THE DEBUT DISCOVERY, THE 5597 03:44:29,120 --> 03:44:30,120 COMPOUND THAT WAS JUST APPROVED, 5598 03:44:30,120 --> 03:44:32,240 IS A PART OF THIS. 5599 03:44:32,240 --> 03:44:34,400 IT'S A SUCCESSFUL STORY FROM A 5600 03:44:34,400 --> 03:44:36,120 PIECE OF THIS. 5601 03:44:36,120 --> 03:44:40,400 WE FUND BASIC RESEARCH, THAT'S 5602 03:44:40,400 --> 03:44:42,600 THE CRITICAL RESEARCH. 5603 03:44:42,600 --> 03:44:44,240 RETT IS THE MOST SEVERE -- ONE 5604 03:44:44,240 --> 03:44:46,000 OF THE MOST SEVERE 5605 03:44:46,000 --> 03:44:46,640 NEURODEVELOPMENTAL DISORDERS 5606 03:44:46,640 --> 03:44:50,680 THAT GIRLS CAN HAVE, IT'S NOT 5607 03:44:50,680 --> 03:44:51,200 NEURODEGENERATIVE. 5608 03:44:51,200 --> 03:44:53,440 BOYS CAN HAVE IT AS WELL. 5609 03:44:53,440 --> 03:44:59,160 PREVALENCE IS ABOUT 1 IN 10,000 5610 03:44:59,160 --> 03:44:59,600 BIRTHS. 5611 03:44:59,600 --> 03:45:01,120 DR. ZOGBE IDENTIFIED THE GENE 5612 03:45:01,120 --> 03:45:03,520 THAT CAUSES RETT SYNDROME, WHEN 5613 03:45:03,520 --> 03:45:09,440 THERE'S A MUTATION OR DELETION, 5614 03:45:09,440 --> 03:45:11,960 MECP2 GENE DISCOVERED IN 1999. 5615 03:45:11,960 --> 03:45:13,600 SINCE 1999 WE'VE BEEN DOING A 5616 03:45:13,600 --> 03:45:17,440 LOT OF WORK TO FURTHER 5617 03:45:17,440 --> 03:45:18,440 UNDERSTAND MECP2 BUT DON'T 5618 03:45:18,440 --> 03:45:20,160 UNDERSTAND IT WELL ENOUGH. 5619 03:45:20,160 --> 03:45:23,720 SO AS A FOUNDATION WE INVEST IN 5620 03:45:23,720 --> 03:45:26,880 CONTINUING TO DEEPEN OUR 5621 03:45:26,880 --> 03:45:29,960 UNDERSTANDING OF WHATTING IT -- 5622 03:45:29,960 --> 03:45:32,160 WHAT IT IS AND HOW IT WORKS 5623 03:45:32,160 --> 03:45:33,760 WHILE INVESTING IN POTENTIAL 5624 03:45:33,760 --> 03:45:34,840 CURATIVE STRATEGIES, GENE 5625 03:45:34,840 --> 03:45:37,480 THERAPY AND OTHER GENETIC 5626 03:45:37,480 --> 03:45:37,800 APPROACHES. 5627 03:45:37,800 --> 03:45:42,680 SIMULTANEOUSLY, WE INVEST IN 5628 03:45:42,680 --> 03:45:43,840 PIPELINE DEVELOPMENT, 5629 03:45:43,840 --> 03:45:44,840 IDENTIFYING MOLECULES, SMALL 5630 03:45:44,840 --> 03:45:45,680 MOLECULES NOT INTENDED 5631 03:45:45,680 --> 03:45:47,280 NECESSARILY TO BE A CURE FOR 5632 03:45:47,280 --> 03:45:53,720 RETT SYNDROME BUT WOULD HAVE A 5633 03:45:53,720 --> 03:45:55,120 MEANINGFUL EFFECT ON DOWNSTREAM 5634 03:45:55,120 --> 03:45:56,400 TARGET INTENDED TO OVERALL 5635 03:45:56,400 --> 03:45:58,720 IMPROVE THE QUALITY OF LIFE BY 5636 03:45:58,720 --> 03:46:01,800 REDUCING MANY, MANY SYMPTOMS OF 5637 03:46:01,800 --> 03:46:03,160 RETT SYNDROME, AND SPOILER 5638 03:46:03,160 --> 03:46:05,120 ALERT, THIS IS WHAT DEBUT IS, 5639 03:46:05,120 --> 03:46:07,120 THE COMPOUND THAT WAS RECENTLY 5640 03:46:07,120 --> 03:46:08,760 APPROVED BY THE FDA. 5641 03:46:08,760 --> 03:46:11,720 AND FINALLY WE ALSO INVEST 5642 03:46:11,720 --> 03:46:13,880 HEAVILY IN A CLINICAL TRIAL 5643 03:46:13,880 --> 03:46:14,600 INFRASTRUCTURE. 5644 03:46:14,600 --> 03:46:16,520 WE SUPPORT CENTER OF EXCELLENCE 5645 03:46:16,520 --> 03:46:16,960 NETWORK. 5646 03:46:16,960 --> 03:46:19,560 WE SUPPORT REGISTRY AS WELL AS 5647 03:46:19,560 --> 03:46:28,800 CONTINUAL WORK AND OUTCOME 5648 03:46:28,800 --> 03:46:29,080 MEASURES. 5649 03:46:29,080 --> 03:46:34,360 IT IS DAYBUE IS THE FIRST 5650 03:46:34,360 --> 03:46:36,000 APPROVED TREATMENT FOR RETT 5651 03:46:36,000 --> 03:46:37,320 SYNDROME, MARCH 10 OF 2023, JUST 5652 03:46:37,320 --> 03:46:41,720 ABOUT A MONTH AFTER RON'S, WHICH 5653 03:46:41,720 --> 03:46:43,040 WAS REALLY EXCITING. 5654 03:46:43,040 --> 03:46:46,960 IT HAPPENED BECAUSE OF REALLY 5655 03:46:46,960 --> 03:46:48,480 HARD WORK, BECAUSE BRILLIANT 5656 03:46:48,480 --> 03:46:50,000 PEOPLE WORKED HARD FOR MANY 5657 03:46:50,000 --> 03:46:51,760 YEARS, IT ALSO HAPPENED BECAUSE 5658 03:46:51,760 --> 03:46:55,480 WE WERE REALLY LUCKY AND THERE 5659 03:46:55,480 --> 03:46:56,720 WAS SERENDIPITY INVOLVED. 5660 03:46:56,720 --> 03:46:58,400 THERE'S BEAUTY IN THAT THAT I'LL 5661 03:46:58,400 --> 03:46:59,080 TALK ABOUT. 5662 03:46:59,080 --> 03:47:03,720 IT IS AS I SAID A SMALL MOLECULE 5663 03:47:03,720 --> 03:47:07,960 INTENDED TO HAVE IMPACT ON 5664 03:47:07,960 --> 03:47:09,400 DOWNSTREAM TARGETS OF THE MECP2 5665 03:47:09,400 --> 03:47:09,840 GENE. 5666 03:47:09,840 --> 03:47:12,120 IN GENERAL I CAN TALK ABOUT 5667 03:47:12,120 --> 03:47:13,320 SWEET ANECDOTES BECAUSE WE ARE 5668 03:47:13,320 --> 03:47:16,240 HAVING MORE AND MORE PEOPLE WHO 5669 03:47:16,240 --> 03:47:18,360 ARE NOW TAKING ADVANTAGE OF 5670 03:47:18,360 --> 03:47:18,600 DAYBUE. 5671 03:47:18,600 --> 03:47:21,280 SOME FRANKLY IT DOESN'T DO A LOT 5672 03:47:21,280 --> 03:47:22,120 FOR. 5673 03:47:22,120 --> 03:47:25,120 THERE ARE OTHERS WHO ARE SEEING 5674 03:47:25,120 --> 03:47:27,320 SUCH A PROFOUND IMPACT, YOUNGER 5675 03:47:27,320 --> 03:47:29,520 INDIVIDUALS, THAT IT'S BEEN 5676 03:47:29,520 --> 03:47:31,480 CALLED DISEASE MODIFYING FOR 5677 03:47:31,480 --> 03:47:32,040 THEM. 5678 03:47:32,040 --> 03:47:34,240 IT IS NOT A CURE BUT IT HAS 5679 03:47:34,240 --> 03:47:37,600 GIVEN US A LOT OF HOPE. 5680 03:47:37,600 --> 03:47:38,560 INTERESTINGLY DAYBUE IS THE 5681 03:47:38,560 --> 03:47:42,200 TRADE NAME FOR A MOLECULE FIRST 5682 03:47:42,200 --> 03:47:52,280 BROUGHT TO THE ATTENTION OF THE 5683 03:47:52,280 --> 03:47:53,360 RETT SYNDROME FOUNDATION, 5684 03:47:53,360 --> 03:47:53,640 TROFINETIDE. 5685 03:47:53,640 --> 03:47:55,800 HE WAS WORKING WITH DoD AND 5686 03:47:55,800 --> 03:47:58,200 SAW THIS MOLECULE COME ACROSS 5687 03:47:58,200 --> 03:47:59,520 HIS DESK AS POTENTIAL TREATMENT 5688 03:47:59,520 --> 03:48:00,480 FOR TRAUMATIC BRAIN INJURY. 5689 03:48:00,480 --> 03:48:02,320 HE LOOKED AT THE MOLECULE AND 5690 03:48:02,320 --> 03:48:04,960 THOUGHT I DON'T KNOW ABOUT TBI 5691 03:48:04,960 --> 03:48:07,160 BUT I THINK THIS MIGHT WORK FOR 5692 03:48:07,160 --> 03:48:08,920 RETT SYNDROME, BROUGHT IT TO THE 5693 03:48:08,920 --> 03:48:10,320 ATTENTION OF OUR FOUNDATION, 5694 03:48:10,320 --> 03:48:12,960 THIS WAS IN 2010 TO 2012. 5695 03:48:12,960 --> 03:48:16,040 OUR FOUNDATION INVESTED IN THE 5696 03:48:16,040 --> 03:48:17,680 MOLECULE, IN THE EARLY CLINICAL 5697 03:48:17,680 --> 03:48:19,200 TRIAL OF THAT MOLECULE, UNTIL IT 5698 03:48:19,200 --> 03:48:22,040 WAS BROUGHT TO A POINT IN PHASE 5699 03:48:22,040 --> 03:48:26,040 3, THAT'S FAR BEYOND WHAT A RARE 5700 03:48:26,040 --> 03:48:31,080 DISEASE FOUNDATION CAN SUPPORT. 5701 03:48:31,080 --> 03:48:33,280 ACADIA PHARMACEUTICALS FUND THE 5702 03:48:33,280 --> 03:48:35,800 PHASE 3 TRIALS AND IT WAS A 5703 03:48:35,800 --> 03:48:36,040 SUCCESS. 5704 03:48:36,040 --> 03:48:37,320 I'M VERY HAPPY TO BE ABLE TO 5705 03:48:37,320 --> 03:48:40,840 SHARE THAT WITH YOU TODAY. 5706 03:48:40,840 --> 03:48:49,920 5707 03:48:49,920 --> 03:48:50,040 5708 03:48:50,040 --> 03:48:52,960 IT'S THEORIZED TO WORK BY 5709 03:48:52,960 --> 03:48:53,640 REDUCING INFLAMMATION, THE 5710 03:48:53,640 --> 03:48:55,280 MECHANISM OF ACTION IS CLEARLY 5711 03:48:55,280 --> 03:48:56,680 NOT DESCRIBED AND DEFINED. 5712 03:48:56,680 --> 03:48:58,800 IN TERMS OF THE CLINICAL TRIAL 5713 03:48:58,800 --> 03:49:01,280 THERE WERE TWO CO-PRIMARY 5714 03:49:01,280 --> 03:49:07,400 ENDPOINTS, ONE CALLED THE RETT 5715 03:49:07,400 --> 03:49:08,960 SYNDROME BEHAVIORAL 5716 03:49:08,960 --> 03:49:09,520 QUESTIONNAIRE, RFBQ, 5717 03:49:09,520 --> 03:49:12,000 CAREGIVER-REPORTED 5718 03:49:12,000 --> 03:49:13,000 QUESTIONNAIRE. 5719 03:49:13,000 --> 03:49:14,360 OTHER IS CGII, CLINICIAN 5720 03:49:14,360 --> 03:49:17,360 ASSESSING GLOBAL IMPROVEMENT. 5721 03:49:17,360 --> 03:49:19,320 THERE WAS STATISTICALLY 5722 03:49:19,320 --> 03:49:21,400 SIGNIFICANT IMPROVEMENT ON BOTH 5723 03:49:21,400 --> 03:49:24,400 SCALES COMPARED TO PLACEBO. 5724 03:49:24,400 --> 03:49:26,400 IT WAS BECAUSE OF THAT THAT A 5725 03:49:26,400 --> 03:49:28,560 DECISION WAS MADE AS WELL AS 5726 03:49:28,560 --> 03:49:30,000 MANY OTHER THINGS, DECISION TO 5727 03:49:30,000 --> 03:49:31,160 APPROVE FOR TREATMENT. 5728 03:49:31,160 --> 03:49:35,040 YOU CAN SEE THERE ARE A GENERAL 5729 03:49:35,040 --> 03:49:44,880 SUBSCALE OF THE RSBQ, IMPROVED 5730 03:49:44,880 --> 03:49:45,840 BREATHING, HAND BEHAVIORS FOR 5731 03:49:45,840 --> 03:49:48,800 THOSE WHO MIGHT NOT BE AWARE, 5732 03:49:48,800 --> 03:49:52,400 RETT SYNDROME HAS A CLASSIC 5733 03:49:52,400 --> 03:49:54,280 MOVE, REPETITIVE HAND MOVEMENT 5734 03:49:54,280 --> 03:49:56,360 AND INDIVIDUALS WITH RETT ARE 5735 03:49:56,360 --> 03:49:58,000 ALMOST ALL UNABLE TO 5736 03:49:58,000 --> 03:50:01,040 MEANINGFULLY USE THEIR HANDS. 5737 03:50:01,040 --> 03:50:04,040 FACE MOVEMENTS, BODY ROCKING, 5738 03:50:04,040 --> 03:50:05,200 EXPRESSIONLESS FACE, NIGHTTIME 5739 03:50:05,200 --> 03:50:06,280 BEHAVIORS, SLEEPING IS A PROBLEM 5740 03:50:06,280 --> 03:50:08,280 FOR FAMILIES WHO HAVE A CHILD 5741 03:50:08,280 --> 03:50:09,160 WITH RETT SYNDROME. 5742 03:50:09,160 --> 03:50:12,680 THERE'S A LOT OF FEAR AND 5743 03:50:12,680 --> 03:50:15,080 ANXIETY THAT IS ASSOCIATED WITH 5744 03:50:15,080 --> 03:50:19,960 RETT, ABILITY TO WALK, 5745 03:50:19,960 --> 03:50:27,040 INDIANENEDLY -- INDEPENDENTLY 5746 03:50:27,040 --> 03:50:28,120 STAND AND MOVE AROUND. 5747 03:50:28,120 --> 03:50:30,520 I'LL TALK ABOUT HOW WE 5748 03:50:30,520 --> 03:50:31,720 INCORPORATED THE FAMILIES INTO 5749 03:50:31,720 --> 03:50:34,280 THE PROCESS AND HOW WE EMBED THE 5750 03:50:34,280 --> 03:50:41,120 FAMILY VOICE IN ALL OF THIS. 5751 03:50:41,120 --> 03:50:42,560 SUCCESSFUL DRUG VALUE. 5752 03:50:42,560 --> 03:50:45,480 HAPPENS BECAUSE IT'S A BEAUTIFUL 5753 03:50:45,480 --> 03:50:48,000 COLLABORATION BETWEEN SCIENTISTS 5754 03:50:48,000 --> 03:50:48,760 AND CLINICIANS AND INDUSTRY'S 5755 03:50:48,760 --> 03:50:50,120 ADVOCACY AND FAMILIES. 5756 03:50:50,120 --> 03:50:52,640 WE DID A FEW THINGS IN THE YEARS 5757 03:50:52,640 --> 03:50:55,760 LEADING UP TO THE FDA APPROVAL. 5758 03:50:55,760 --> 03:51:01,360 WE DID HOLD AN EXTERNALLY LED 5759 03:51:01,360 --> 03:51:05,200 MEETING IN MARCH OF 2020, A VERY 5760 03:51:05,200 --> 03:51:06,440 SUCCESSFUL MEETING, TIMING WAS 5761 03:51:06,440 --> 03:51:07,160 PERFECT. 5762 03:51:07,160 --> 03:51:12,960 OVER THE NEXT YEAR THE FDA 5763 03:51:12,960 --> 03:51:14,280 REVIEWED TROFINETIDE FOR 5764 03:51:14,280 --> 03:51:18,240 POTENTIAL APPROVAL AND HAD THE 5765 03:51:18,240 --> 03:51:20,320 EXTERNALLY LED PATIENT-FOCUSED 5766 03:51:20,320 --> 03:51:21,200 DRUG DEVELOPMENT MEETING REPORT 5767 03:51:21,200 --> 03:51:23,280 AVAILABLE AS PART OF THE REVIEW. 5768 03:51:23,280 --> 03:51:27,560 WE LAUNCHED WHAT WE CALL RETT 5769 03:51:27,560 --> 03:51:28,600 RESEARCH READY, OUR ATTEMPT TO 5770 03:51:28,600 --> 03:51:29,960 HELP FAMILIES BECOME RESEARCH 5771 03:51:29,960 --> 03:51:34,520 PARTNERS, LIVES ON OUR WEBSITE, 5772 03:51:34,520 --> 03:51:35,600 TEACHES FAMILIES WHAT 5773 03:51:35,600 --> 03:51:36,760 PARTICIPATION IN CLINICAL 5774 03:51:36,760 --> 03:51:40,360 RESEARCH LOOKS LIKE AND ALSO 5775 03:51:40,360 --> 03:51:42,000 INCORPORATION MY TRIAL FINDER SO 5776 03:51:42,000 --> 03:51:44,160 FAMILIES CAN FIND TRIALS CLOSE 5777 03:51:44,160 --> 03:51:45,040 TO THEM. 5778 03:51:45,040 --> 03:51:46,040 WE'VE LAUNCHED CLINICAL TRIAL 5779 03:51:46,040 --> 03:51:50,880 COMMITTEE WHICH IS A GROUP OF 5780 03:51:50,880 --> 03:51:53,400 KOLs THAT ADVISE INDUSTRY ON 5781 03:51:53,400 --> 03:51:55,040 PROTOCOL DESIGN AND OTHER 5782 03:51:55,040 --> 03:51:57,200 THINGS, AND WE HAVE FAMILIES 5783 03:51:57,200 --> 03:51:59,080 THAT HAVE A SEAT AT THAT TABLE. 5784 03:51:59,080 --> 03:52:00,960 SO THEY ARE PARTICIPATING IN THE 5785 03:52:00,960 --> 03:52:02,680 CONVERSATION WITH INDUSTRY AND 5786 03:52:02,680 --> 03:52:05,160 WITH OUR CLINICIANS AND KOLs 5787 03:52:05,160 --> 03:52:08,040 WHEN PROTOCOL DESIGN IS 5788 03:52:08,040 --> 03:52:08,320 DISCUSSED. 5789 03:52:08,320 --> 03:52:10,200 I'M GOING TO STOP THERE SO WE 5790 03:52:10,200 --> 03:52:11,760 HAVE TIME FOR EVERYBODY ELSE, 5791 03:52:11,760 --> 03:52:12,960 BUT I REALLY APPRECIATE THE 5792 03:52:12,960 --> 03:52:13,960 OPPORTUNITY TO SPEAK WITH YOU 5793 03:52:13,960 --> 03:52:14,200 TODAY. 5794 03:52:14,200 --> 03:52:22,480 THANK YOU. 5795 03:52:22,480 --> 03:52:27,400 5796 03:52:27,400 --> 03:52:30,120 >> I'M STILL RON BARTEK. 5797 03:52:30,120 --> 03:52:31,960 I'M HONORED TO BE A MEMBER OF 5798 03:52:31,960 --> 03:52:33,400 THIS PANEL AND REALLY THRILLED 5799 03:52:33,400 --> 03:52:35,600 TO BE A MEMBER OF ANYTHING 5800 03:52:35,600 --> 03:52:39,080 CALLED A SUCCESS STORY PANEL. 5801 03:52:39,080 --> 03:52:45,480 FOR THE FIRST TIME IN 25 YEARS, 5802 03:52:45,480 --> 03:52:45,680 YEAH. 5803 03:52:45,680 --> 03:52:46,040 SO ... 5804 03:52:46,040 --> 03:52:47,800 I'LL GET STARTED ON THAT 5805 03:52:47,800 --> 03:52:48,360 EMOTIONAL NOTE. 5806 03:52:48,360 --> 03:52:53,440 I WANT TO TELL YOU JUST ENOUGH 5807 03:52:53,440 --> 03:52:56,200 ABOUT FRIEDRICH'S ATAXIA SO KNOW 5808 03:52:56,200 --> 03:52:57,280 WHAT YOU DRUG SEEMS TO BE DOING 5809 03:52:57,280 --> 03:52:58,600 AND HOW. 5810 03:52:58,600 --> 03:53:02,680 IT'S A RARE RECESSIVE TRAIT, 5811 03:53:02,680 --> 03:53:03,320 GENETIC NEUROMUSCULAR DISEASE, 5812 03:53:03,320 --> 03:53:06,800 5,000 PEOPLE IN THE U.S. ARE 5813 03:53:06,800 --> 03:53:07,080 AFFECTED. 5814 03:53:07,080 --> 03:53:14,840 AND MAYBE 20,000 WORLDWIDE. 5815 03:53:14,840 --> 03:53:16,040 RESULTS FROM A TRINUCLEOTIDE 5816 03:53:16,040 --> 03:53:22,840 EXPANSION AND THE FIRST INTRON 5817 03:53:22,840 --> 03:53:23,840 OF A GENE. 5818 03:53:23,840 --> 03:53:27,680 IF THE MUTATION IS IN AN INTRON 5819 03:53:27,680 --> 03:53:30,080 YOU WOULDN'T EXPECT IT TO AFFECT 5820 03:53:30,080 --> 03:53:32,160 A PROTEIN, RIGHT? 5821 03:53:32,160 --> 03:53:35,120 IT'S THE EXONS THAT GENERATE THE 5822 03:53:35,120 --> 03:53:36,000 PROTEIN. 5823 03:53:36,000 --> 03:53:40,320 BUT IN THIS CASE, THE EFFECT OF 5824 03:53:40,320 --> 03:53:43,880 THE INTRONIC EXPANSION UP TO A 5825 03:53:43,880 --> 03:53:46,160 THOUSAND OR SO, 1500 GAA 5826 03:53:46,160 --> 03:53:49,000 REPEATS, IS TO PARTIALLY SILENCE 5827 03:53:49,000 --> 03:53:50,320 THAT GENE THROUGH MECHANISMS 5828 03:53:50,320 --> 03:53:53,480 THAT WE THINK WE NOW FULLY 5829 03:53:53,480 --> 03:53:53,800 UNDERSTAND. 5830 03:53:53,800 --> 03:53:58,440 SO, WHAT YOU DO IS YOU REDUCE, 5831 03:53:58,440 --> 03:54:00,600 PARTIALLY SILENCING EXPRESSION 5832 03:54:00,600 --> 03:54:03,040 OF PROTEIN, REDUCING PROTEIN 5833 03:54:03,040 --> 03:54:06,080 EXPRESSION FROM 100% IN OUR 5834 03:54:06,080 --> 03:54:07,960 NORMALS TO ABOUT 10 TO 15% IN 5835 03:54:07,960 --> 03:54:08,920 OUR PATIENTS. 5836 03:54:08,920 --> 03:54:11,040 GOOD NEWS HERE TOO THOUGH, THEIR 5837 03:54:11,040 --> 03:54:12,760 PARENTS ARE CARRIERS OF THAT 5838 03:54:12,760 --> 03:54:15,840 MUTATION, SO THEY ARE ONLY 5839 03:54:15,840 --> 03:54:18,680 GETTING 40 TO 60% OF THAT 5840 03:54:18,680 --> 03:54:22,480 PROTECTION PROTEIN, AND THEY ARE 5841 03:54:22,480 --> 03:54:23,600 COMPLETELY HEALTHY. 5842 03:54:23,600 --> 03:54:27,080 THAT'S THE TARGET SET THAT WE 5843 03:54:27,080 --> 03:54:28,000 STARTED WITH. 5844 03:54:28,000 --> 03:54:31,120 AND WHAT'S THE IMPACT OF THOSE 5845 03:54:31,120 --> 03:54:33,280 LOW LEVELS OF PROTEIN? 5846 03:54:33,280 --> 03:54:34,720 WELL, IN TWO WORDS, 5847 03:54:34,720 --> 03:54:35,600 MITOCHONDRIAL DYSFUNCTION. 5848 03:54:35,600 --> 03:54:37,680 MOST OF YOU KNOW THAT THE 5849 03:54:37,680 --> 03:54:38,680 MITOCHONDRIA ARE THE POWER 5850 03:54:38,680 --> 03:54:42,520 PLANTS OF ALL OF OUR CELLS, 5851 03:54:42,520 --> 03:54:44,000 RESPONSIBLE FOR PRODUCING ATP, 5852 03:54:44,000 --> 03:54:46,480 THE CURRENCY OF ENERGY FOR OUR 5853 03:54:46,480 --> 03:54:46,680 CELLS. 5854 03:54:46,680 --> 03:54:52,000 AND ON THE SIDE PRODUCE WATER. 5855 03:54:52,000 --> 03:54:53,960 NET RESULT OF HEALTHY 5856 03:54:53,960 --> 03:54:57,600 MITOCHONDRIA IS ATP AND WATER. 5857 03:54:57,600 --> 03:55:00,880 THE FUNCTION OF THING PROTEIN IS 5858 03:55:00,880 --> 03:55:03,520 TO GENERAL -- GENERATED IN THE 5859 03:55:03,520 --> 03:55:06,600 NUCLEUS, GOES INTO CYTOPLASM, 5860 03:55:06,600 --> 03:55:08,000 BINDS WITH IRON, KEEPS IRON IN 5861 03:55:08,000 --> 03:55:18,400 PROPER STATE TO MOVE TO 5862 03:55:21,320 --> 03:55:22,360 MITOCHONDRIA MATRICES, TO 5863 03:55:22,360 --> 03:55:24,760 PRODUCE DOWN AT THE END THE 5864 03:55:24,760 --> 03:55:27,920 FIFTH COMPLEX, YOU GET ATP AND 5865 03:55:27,920 --> 03:55:28,120 WATER. 5866 03:55:28,120 --> 03:55:32,080 IF YOU HAVE A SHORT SUPPLY OF 5867 03:55:32,080 --> 03:55:34,040 THE PROTEIN IT DOESN'T BIND WITH 5868 03:55:34,040 --> 03:55:38,040 ENOUGH IRON TO JOIN WITH THE 5869 03:55:38,040 --> 03:55:41,880 IRON SULPHUR CLUSTERS, SO IT 5870 03:55:41,880 --> 03:55:44,440 FORMS FERROUS OXIDE, LOSES -- 5871 03:55:44,440 --> 03:55:47,800 INSTEAD OF ESCORTING ELECTRONS 5872 03:55:47,800 --> 03:55:52,160 WITH THE CLUSTERS ALONG THE 5873 03:55:52,160 --> 03:55:58,400 MATRICES THEY ESCAPE AND FORM 5874 03:55:58,400 --> 03:55:58,680 RADICALS. 5875 03:55:58,680 --> 03:56:03,360 NET RESULT OF LOW LEVELS IS 5876 03:56:03,360 --> 03:56:05,000 INCREASED OXIDATIVE STRESS, 5877 03:56:05,000 --> 03:56:05,520 INCREASED INFLAMMATION, 5878 03:56:05,520 --> 03:56:07,200 DECREASED ENERGY PRODUCTION. 5879 03:56:07,200 --> 03:56:10,160 IT'S A DOUBLE WHAMMY IN THIS 5880 03:56:10,160 --> 03:56:14,760 CASE BECAUSE AS THE LOW LEVELS 5881 03:56:14,760 --> 03:56:17,080 OF FURTAXIN ARE CAUSING 5882 03:56:17,080 --> 03:56:19,280 INFLAMMATION, GREATER OXIDATIVE 5883 03:56:19,280 --> 03:56:24,800 STRESS, NATURAL DEFENSES OF A 5884 03:56:24,800 --> 03:56:29,840 HEALTHY CELL NAMELY Nrf2 5885 03:56:29,840 --> 03:56:31,480 TRANSCRIPTION FACTOR, ITS 5886 03:56:31,480 --> 03:56:38,800 RESPONSIBILITY IS ACTIVATE GENES 5887 03:56:38,800 --> 03:56:44,640 THAT PRODUCE THE TO COMBAT 5888 03:56:44,640 --> 03:56:45,720 OXIDATIVE STRESS, COMBAT 5889 03:56:45,720 --> 03:56:47,480 INFLAMMATION. 5890 03:56:47,480 --> 03:56:52,160 YOU EXPECT LOW LEVELS OF 5891 03:56:52,160 --> 03:56:55,280 FURTAXIN, MORE NRF2. 5892 03:56:55,280 --> 03:56:58,000 BOLOGNA. 5893 03:56:58,000 --> 03:57:02,160 COUNTER INTUITIVELY, AS YOU 5894 03:57:02,160 --> 03:57:05,120 DROPPED FURTAXIN, THE NRF2 5895 03:57:05,120 --> 03:57:08,840 PATHWAY IS DEMOBILIZED. 5896 03:57:08,840 --> 03:57:10,280 LESS ENERGY, AND WE CAN'T RALLY 5897 03:57:10,280 --> 03:57:15,760 THE TROOPS OF OUR NORMAL 5898 03:57:15,760 --> 03:57:21,080 DEFENSES AGAINST THAT 5899 03:57:21,080 --> 03:57:23,640 INFLAMMATION AND OXIDATIVE 5900 03:57:23,640 --> 03:57:24,080 STRESS. 5901 03:57:24,080 --> 03:57:29,200 NET RESULT IT'S PROGRESSIVE, 5902 03:57:29,200 --> 03:57:34,040 DIAGNOSED BY 7 AND 15, IMPACTS 5903 03:57:34,040 --> 03:57:39,720 VISION, HEARING, SPEECH, SEVERE 5904 03:57:39,720 --> 03:57:40,680 SCOLIOSIS REGARDING SURGICAL 5905 03:57:40,680 --> 03:57:42,560 IMPLANTATION OF METAL RODS TO 5906 03:57:42,560 --> 03:57:44,520 KEEP THE SPINE STRAIGHT, MUCH 5907 03:57:44,520 --> 03:57:47,800 INCREASED RISK OF DIABETES AND 5908 03:57:47,800 --> 03:57:49,640 MOST CONDEMNING FACTOR IS HEART 5909 03:57:49,640 --> 03:57:52,120 CONDITION THAT LEADS TO 5910 03:57:52,120 --> 03:57:53,040 CONGESTIVE HEART FAILURE. 5911 03:57:53,040 --> 03:58:03,520 AND THE END OF YOUNG LIVES. 5912 03:58:12,040 --> 03:58:14,600 5913 03:58:14,600 --> 03:58:17,360 ENTER PHARMACEUTICALS. 5914 03:58:17,360 --> 03:58:18,120 NOW CALLED SKYCLARYS. 5915 03:58:18,120 --> 03:58:20,520 WHAT DOES IT DO? 5916 03:58:20,520 --> 03:58:23,160 WELL, IT MOVES INTO THE ARENA IN 5917 03:58:23,160 --> 03:58:25,560 WHICH WE'VE JUST DESCRIBED. 5918 03:58:25,560 --> 03:58:28,400 RISING STRESS AND INFLAMMATION. 5919 03:58:28,400 --> 03:58:30,800 AND LOW ENERGY. 5920 03:58:30,800 --> 03:58:33,880 THE MITOCHONDRIA ARE STRUGGLING 5921 03:58:33,880 --> 03:58:39,120 AND DYING, WHICH LEADS TO 5922 03:58:39,120 --> 03:58:40,400 APOPTOSIS, CELL DEATH. 5923 03:58:40,400 --> 03:58:44,240 AND THE ALERT GOES OUT THAT'S A 5924 03:58:44,240 --> 03:58:46,440 LITTLE MITOCHONDRION, BY THE 5925 03:58:46,440 --> 03:58:48,000 WAY, THE SQUIGGLY LINE TO THE 5926 03:58:48,000 --> 03:58:48,840 UPPER LEFT. 5927 03:58:48,840 --> 03:58:55,080 THEY ARE IN DISTRESS, 5928 03:58:55,080 --> 03:58:55,840 STRUGGLING. 5929 03:58:55,840 --> 03:58:58,160 THEY ARE NOT GETTING NORMAL 5930 03:58:58,160 --> 03:59:03,280 SUPPORT FROM NORMAL DEFENSES. 5931 03:59:03,280 --> 03:59:05,280 ENTER OMAV, MADE FROM FROM AN 5932 03:59:05,280 --> 03:59:08,560 ACID AVAILABLE IN VARIOUS 5933 03:59:08,560 --> 03:59:09,120 PLANTS. 5934 03:59:09,120 --> 03:59:14,120 MECHANISM OF ACTION IS TO 5935 03:59:14,120 --> 03:59:18,320 ACTUALLY REACTIVATE THE NRF2 5936 03:59:18,320 --> 03:59:19,280 PATHWAY, TO GENERATE DEFENSE, 5937 03:59:19,280 --> 03:59:23,000 NORMAL DEFENSES TO A LARGE 5938 03:59:23,000 --> 03:59:24,000 EXTENT. 5939 03:59:24,000 --> 03:59:26,840 AND IT WILL SEND THOSE 5940 03:59:26,840 --> 03:59:29,040 ACTIVATORS TO PRODUCE THE 5941 03:59:29,040 --> 03:59:30,840 DEFENSES AND PROTECT FIRST THE 5942 03:59:30,840 --> 03:59:39,480 MITOCHONDRIA, AND THEN THE WHOLE 5943 03:59:39,480 --> 03:59:43,880 CELL. 5944 03:59:43,880 --> 03:59:45,520 MORE ENERGY, LESS INFLAMMATION, 5945 03:59:45,520 --> 03:59:46,520 LESS OXIDATIVE STRESS, WHAT 5946 03:59:46,520 --> 03:59:48,720 CONVINCED US AND THE FDA THIS 5947 03:59:48,720 --> 03:59:49,760 DRUG WAS GOING TO WORK? 5948 03:59:49,760 --> 03:59:58,000 WHAT YOU SEE ON THE LEFT, LET'S 5949 03:59:58,000 --> 03:59:59,320 MAKE THIS WORK. 5950 03:59:59,320 --> 04:00:03,400 YOU SEE RIGHT THERE THESE ARE 5951 04:00:03,400 --> 04:00:05,160 THE DATA RESULTS OF PHASE 2 5952 04:00:05,160 --> 04:00:06,120 CLINICAL TRIAL. 5953 04:00:06,120 --> 04:00:13,560 ON THE TOP YOU SEE THE CONTROL 5954 04:00:13,560 --> 04:00:13,760 GROUP. 5955 04:00:13,760 --> 04:00:19,120 BOTTOM BLUE LINE YOU SEE THE 5956 04:00:19,120 --> 04:00:20,120 ACTIVE GROUP. 5957 04:00:20,120 --> 04:00:24,360 YOU SEE THESE DATA POINTS ARE ON 5958 04:00:24,360 --> 04:00:26,360 OUR FRIEDRICH'S ATAXIA RATING 5959 04:00:26,360 --> 04:00:28,440 SCHOOL, LIKE GOLF, LOW SCORE 5960 04:00:28,440 --> 04:00:29,320 WINS. 5961 04:00:29,320 --> 04:00:30,720 IF YOU'RE BEING EFFECTIVELY 5962 04:00:30,720 --> 04:00:37,640 TREATED, YOUR SCORE IS GOING 5963 04:00:37,640 --> 04:00:37,920 DOWN. 5964 04:00:37,920 --> 04:00:41,360 IF NOT EFFECTIVELY TREATED SCORE 5965 04:00:41,360 --> 04:00:42,120 GOES UP. 5966 04:00:42,120 --> 04:00:46,080 YOU CAN SEE THE DIFFERENCE 5967 04:00:46,080 --> 04:00:51,520 THERE, THE CONTROL GROUP IS 5968 04:00:51,520 --> 04:00:53,120 GETTING WORSE THROUGHOUT THE 5969 04:00:53,120 --> 04:00:55,280 YEAR, 48 WEEKS OF THE TRIAL. 5970 04:00:55,280 --> 04:00:57,240 THE ACTIVE GROUP IS ACTUALLY 5971 04:00:57,240 --> 04:00:57,520 IMPROVING. 5972 04:00:57,520 --> 04:00:58,760 THAT'S NEVER HAPPENED TO OUR 5973 04:00:58,760 --> 04:01:00,400 PATIENTS BEFORE. 5974 04:01:00,400 --> 04:01:02,400 THEY ACTUALLY IMPROVED. 5975 04:01:02,400 --> 04:01:03,880 IT WASN'T JUST THAT THEY WERE 5976 04:01:03,880 --> 04:01:06,320 DOING BETTER THAN THE CONTROL 5977 04:01:06,320 --> 04:01:08,200 GROUP. 5978 04:01:08,200 --> 04:01:10,880 THEY WERE ACTUALLY IMPROVING. 5979 04:01:10,880 --> 04:01:12,800 NET RESULT WAS 2.4-POINT 5980 04:01:12,800 --> 04:01:21,320 DIFFERENCE ON RATING SCALE. 5981 04:01:21,320 --> 04:01:23,520 SO, THE OTHER DATA POINT, AND 5982 04:01:23,520 --> 04:01:26,160 THIS IS DIRECT RESULT OF OUR 5983 04:01:26,160 --> 04:01:27,280 NATURAL HISTORY DATABASE, THAT 5984 04:01:27,280 --> 04:01:32,080 I'LL TALK MORE ABOUT LATER, 5985 04:01:32,080 --> 04:01:38,560 AFTER THAT PHASE 2 WAS DONE, 5986 04:01:38,560 --> 04:01:44,560 THAT ONE, THE REATTA 5987 04:01:44,560 --> 04:01:46,760 PHARMACEUTICALS GATHERED 5988 04:01:46,760 --> 04:01:49,320 PERFECTLY MATCHED COHORT OF 136 5989 04:01:49,320 --> 04:01:50,360 PATIENTS, COMPARED TO 136 5990 04:01:50,360 --> 04:01:55,720 PATIENTS IN THE OPEN LABEL 5991 04:01:55,720 --> 04:01:58,760 EXTENSION ACROSS THREE YEARS. 5992 04:01:58,760 --> 04:02:01,080 THEY SAW THE -- NOW, EVERYBODY 5993 04:02:01,080 --> 04:02:04,560 IN THE OPEN LABEL EXTENSION IS 5994 04:02:04,560 --> 04:02:05,280 ON DRUG. 5995 04:02:05,280 --> 04:02:12,600 SO THAT'S THIS BLUE LINE. 5996 04:02:12,600 --> 04:02:14,360 TOP LINE IS CAREFULLY MATCHED 5997 04:02:14,360 --> 04:02:15,840 136 PATIENTS ON NO DRUG, DIDN'T 5998 04:02:15,840 --> 04:02:16,560 HAVE ANY. 5999 04:02:16,560 --> 04:02:18,160 LOOK AT THE DIFFERENCE. 6000 04:02:18,160 --> 04:02:21,040 WHAT THAT REPRESENTS IS A 55% 6001 04:02:21,040 --> 04:02:22,560 SLOWING OF PROGRESSION IN OUR 6002 04:02:22,560 --> 04:02:23,520 DISEASE ACROSS THOSE THREE 6003 04:02:23,520 --> 04:02:23,960 YEARS. 6004 04:02:23,960 --> 04:02:28,320 IF YOU TOOK IT BACK FURTHER, TWO 6005 04:02:28,320 --> 04:02:31,080 YEARS OF THE PHASE 2 TRIAL, IT 6006 04:02:31,080 --> 04:02:34,120 WOULD BE MORE THAN 55% SLOWING 6007 04:02:34,120 --> 04:02:36,000 OF PROGRESSION. 6008 04:02:36,000 --> 04:02:42,000 SO THAT GOT US EXCITED. 6009 04:02:42,000 --> 04:02:44,840 AND LET'S TALK ABOUT HOW WE GOT 6010 04:02:44,840 --> 04:02:45,200 THERE. 6011 04:02:45,200 --> 04:02:54,040 HOW DID WE GET TO THAT KIND OF 6012 04:02:54,040 --> 04:02:54,480 RESULT? 6013 04:02:54,480 --> 04:02:56,560 STARTING IN 2009, GOING BACK TO 6014 04:02:56,560 --> 04:02:57,560 SUPPORTING BASIC RESEARCH. 6015 04:02:57,560 --> 04:03:00,280 WE SPENT THE FIRST FEW YEARS OF 6016 04:03:00,280 --> 04:03:02,520 OUR EXISTENCE SUPPORTING 6017 04:03:02,520 --> 04:03:03,720 PRIMARILY BASIC RESEARCH. 6018 04:03:03,720 --> 04:03:07,200 ONE OF THOSE BASIC RESEARCHERS 6019 04:03:07,200 --> 04:03:10,560 HAD FIRST IDENTIFIED IN 2009 6020 04:03:10,560 --> 04:03:13,760 THAT Nrf2 PATHWAY, AS 6021 04:03:13,760 --> 04:03:14,360 IMPORTANT, POTENTIALLY IMPORTANT 6022 04:03:14,360 --> 04:03:14,960 IN FA. 6023 04:03:14,960 --> 04:03:18,240 THAT WAS A GROUP IN FRANCE. 6024 04:03:18,240 --> 04:03:26,480 A FEW YEARS LATER 2013 A 6025 04:03:26,480 --> 04:03:28,640 CALIFORNIA GROUP SHOWED IT WAS 6026 04:03:28,640 --> 04:03:35,240 ADDRESSING THE PATHOPHYSIOLOGY 6027 04:03:35,240 --> 04:03:37,960 OF THE MOUSE. 6028 04:03:37,960 --> 04:03:40,480 WE HEARD FROM A FAMILY, TWO 6029 04:03:40,480 --> 04:03:44,840 PARENTS OF AN FA CHILD, CALLEDS 6030 04:03:44,840 --> 04:03:46,720 AND SAID -- THE WIFE SAID MY 6031 04:03:46,720 --> 04:03:54,280 HUSBAND HAS BEEN WORKING TANG 6032 04:03:54,280 --> 04:03:59,240 TANGENTALLY WITH A DRUG COMPANY, 6033 04:03:59,240 --> 04:04:00,960 REATA, I SEE IN TWO RESEARCH 6034 04:04:00,960 --> 04:04:03,280 STUDIES IN FRANCE AND CALIFORNIA 6035 04:04:03,280 --> 04:04:06,080 THAT THE NRF2 PATHWAY IS RELATED 6036 04:04:06,080 --> 04:04:07,280 TO FRIEDRICH'S ATAXIA, MAYBE WE 6037 04:04:07,280 --> 04:04:11,920 OUGHT TO LOOK INTO THAT. 6038 04:04:11,920 --> 04:04:13,560 OUR BEST CLINICIAN, MYSELF AND 6039 04:04:13,560 --> 04:04:15,640 EXECUTIVE DIRECTOR FLEW TO 6040 04:04:15,640 --> 04:04:18,280 DALLAS, PITCHED THE COMPANY, 6041 04:04:18,280 --> 04:04:20,040 THEY SAID, YEAH, MIGHT BE 6042 04:04:20,040 --> 04:04:22,640 INTERESTING BUT WE'RE WORKING ON 6043 04:04:22,640 --> 04:04:26,400 A DIFFERENT DISEASE. 6044 04:04:26,400 --> 04:04:29,080 SO THEY DEMURRED. 6045 04:04:29,080 --> 04:04:30,320 LATER THE NEXT YEAR, THEY CALLED 6046 04:04:30,320 --> 04:04:32,480 US BACK AND SAID, OKAY, WE'VE 6047 04:04:32,480 --> 04:04:32,920 RECONSIDERED. 6048 04:04:32,920 --> 04:04:35,880 WE THINK WE HAVE A CANDIDATE 6049 04:04:35,880 --> 04:04:40,280 THAT MIGHT BE OF INTEREST TO THE 6050 04:04:40,280 --> 04:04:41,680 FA COMMUNITY, LET'S START 6051 04:04:41,680 --> 04:04:41,920 MEETING. 6052 04:04:41,920 --> 04:04:52,120 THEY GOT TOGETHER WITH OUR BEST 6053 04:04:52,120 --> 04:04:52,880 CLINICIANS, FARA LEADERSHIP, 6054 04:04:52,880 --> 04:04:55,280 USED OUR NATURAL HISTORY STUDY 6055 04:04:55,280 --> 04:04:56,400 TO PLAN THEIR FIRST CLINICAL 6056 04:04:56,400 --> 04:04:56,680 TRIAL. 6057 04:04:56,680 --> 04:04:58,360 AND THEY USED THE NATURAL 6058 04:04:58,360 --> 04:05:00,880 HISTORY STUDY AND OUR BEST 6059 04:05:00,880 --> 04:05:03,840 CLINICIANS AND LEADERSHIP TO 6060 04:05:03,840 --> 04:05:05,600 DESIGN THAT CLINICAL TRIAL 6061 04:05:05,600 --> 04:05:08,680 AROUND ENDPOINTS THAT WE 6062 04:05:08,680 --> 04:05:09,760 DEVELOPED, AND HOLD THAT 6063 04:05:09,760 --> 04:05:11,080 CLINICAL TRIAL AT CLINICAL SITES 6064 04:05:11,080 --> 04:05:16,120 WE WERE SUPPORTING FOR YEARS. 6065 04:05:16,120 --> 04:05:17,400 WITH PHYSICIANS THAT KNEW OUR 6066 04:05:17,400 --> 04:05:18,840 PATIENTS AND OUR DISEASE. 6067 04:05:18,840 --> 04:05:21,240 TWO YEARS LATER FINISHED THAT 6068 04:05:21,240 --> 04:05:23,320 TRIAL, IT WAS SUFFICIENTLY 6069 04:05:23,320 --> 04:05:24,600 PROMISING RESULTS, THAT THEY 6070 04:05:24,600 --> 04:05:28,000 STARTED A SECOND CLINICAL TRIAL, 6071 04:05:28,000 --> 04:05:31,760 SAME CLINICAL SITES WE'VE BEEN 6072 04:05:31,760 --> 04:05:35,040 SUPPORTING FOR 15 YEARS. 6073 04:05:35,040 --> 04:05:37,320 IN 2020 AFTER FINISHING THAT 6074 04:05:37,320 --> 04:05:38,640 SECOND TRIAL, BEING VERY 6075 04:05:38,640 --> 04:05:40,880 ENCOURAGED BY THE RESULTS, WE 6076 04:05:40,880 --> 04:05:43,080 HAD MULTIPLE MEETINGS WITH THE 6077 04:05:43,080 --> 04:05:44,000 FDA. 6078 04:05:44,000 --> 04:05:45,960 FARA ALWAYS INVITED TO THESE, 6079 04:05:45,960 --> 04:05:50,240 PLAYING AN INTEGRAL ROLE IN THE 6080 04:05:50,240 --> 04:05:52,080 DISCUSSION. 6081 04:05:52,080 --> 04:05:55,680 2021, AFTER HEARING -- AFTER 6082 04:05:55,680 --> 04:05:58,440 MULTIPLE MEETINGS THE FDA 6083 04:05:58,440 --> 04:06:01,960 REVIEWERS WERE NOTE -- DID NOT 6084 04:06:01,960 --> 04:06:05,320 FIND DATA PERSUASIVE, WANTED US 6085 04:06:05,320 --> 04:06:06,400 TO DO ANOTHER PHASE 2 CLINICAL 6086 04:06:06,400 --> 04:06:06,720 TRIAL. 6087 04:06:06,720 --> 04:06:09,040 WE LAUNCHED A PUBLIC APPEAL, GOT 6088 04:06:09,040 --> 04:06:11,600 MORE THAN 74,000 SIGNATURES FROM 6089 04:06:11,600 --> 04:06:13,120 OUR PATIENTS, THEIR FAMILIES, 6090 04:06:13,120 --> 04:06:17,080 OUR BEST CLINICIANS, BEST BASIC 6091 04:06:17,080 --> 04:06:18,680 SCIENTISTS, AND FARA LEADERSHIP, 6092 04:06:18,680 --> 04:06:19,600 ALL IN DEMONSTRATING IT WASN'T 6093 04:06:19,600 --> 04:06:21,000 THROW THE BLOOD ON THE WALL. 6094 04:06:21,000 --> 04:06:26,360 IT WAS LIKE WE'RE ALL CONVINCED 6095 04:06:26,360 --> 04:06:28,240 THESE CLINICAL TRIAL RESULTS ARE 6096 04:06:28,240 --> 04:06:30,600 SUFFICIENTLY PERSUASIVE, ARE 6097 04:06:30,600 --> 04:06:32,800 GOING TO LEAD TO CLINICAL 6098 04:06:32,800 --> 04:06:33,480 BENEFIT. 6099 04:06:33,480 --> 04:06:36,200 THE FDA AGREED THEY WOULD 6100 04:06:36,200 --> 04:06:38,280 RECEIVE AND REVIEW A NEW DRUG 6101 04:06:38,280 --> 04:06:39,480 APPLICATION FOR THE COMPANY 6102 04:06:39,480 --> 04:06:41,200 BASED ON THAT APPEAL BECAUSE IT 6103 04:06:41,200 --> 04:06:43,080 SHOWED CONSENSUS. 6104 04:06:43,080 --> 04:06:44,720 THERE WAS NO DEBATE. 6105 04:06:44,720 --> 04:06:47,240 EVERYBODY IN OUR COMMUNITY, THE 6106 04:06:47,240 --> 04:06:48,120 BEST CLINICIANS THAT HAD BEEN 6107 04:06:48,120 --> 04:06:49,880 SEEING THESE PATIENTS FOR 6108 04:06:49,880 --> 04:06:53,040 DECADES, AND SO FORTH, AGREE 6109 04:06:53,040 --> 04:06:56,560 THIS COULD BE CLINICALLY 6110 04:06:56,560 --> 04:06:56,880 BENEFICIAL. 6111 04:06:56,880 --> 04:06:58,720 SO, THEY SAID ON THE BASIS OF 6112 04:06:58,720 --> 04:07:02,160 THAT APPEAL WE'LL GO AHEAD AND 6113 04:07:02,160 --> 04:07:07,320 RECEIVE AND REVIEW YOUR NDA. 6114 04:07:07,320 --> 04:07:09,240 AND REATA COMPLETED THAT ROLLING 6115 04:07:09,240 --> 04:07:12,120 SUBMISSION AS A RESULT OF HAVING 6116 04:07:12,120 --> 04:07:15,640 FAST TRACK STATUS, IN MARCH OF 6117 04:07:15,640 --> 04:07:17,920 LAST YEAR. 6118 04:07:17,920 --> 04:07:19,800 AND THEN THE LAST SUMMER THEY 6119 04:07:19,800 --> 04:07:25,480 SUBMITTED THE DATA THAT I'VE 6120 04:07:25,480 --> 04:07:29,400 ALREADY SHOWN YOU THREE 6121 04:07:29,400 --> 04:07:30,080 ADDITIONAL DATASETS 6122 04:07:30,080 --> 04:07:30,680 SUPPLEMENTING THE OTHERWISE 6123 04:07:30,680 --> 04:07:31,600 POSITIVE CLINICAL TRIAL RESULTS 6124 04:07:31,600 --> 04:07:33,960 FROM THE SINGLE CLINICAL TRIAL, 6125 04:07:33,960 --> 04:07:35,000 USING OUR NATURAL HISTORY 6126 04:07:35,000 --> 04:07:38,800 DATABASE, FOR THE FIRST TIME IN 6127 04:07:38,800 --> 04:07:40,760 FDA HISTORY, THEY LOOKED AT 6128 04:07:40,760 --> 04:07:45,480 NATURAL HISTORY DATA AS AN 6129 04:07:45,480 --> 04:07:48,440 EXTERNAL CONTROL TO PROVIDE 6130 04:07:48,440 --> 04:07:50,000 CONFIRMATORY EVIDENCE OF 6131 04:07:50,000 --> 04:07:53,280 OTHERWISE POSITIVE SINGLE-TRIAL 6132 04:07:53,280 --> 04:07:53,720 DATA. 6133 04:07:53,720 --> 04:07:56,800 AND THAT LED TO ON RARE DISEASE 6134 04:07:56,800 --> 04:08:02,520 DAY OF THIS YEAR RIGHT 6135 04:08:02,520 --> 04:08:08,080 DOWNSTAIRS IN THE MASUR 6136 04:08:08,080 --> 04:08:09,720 AUDITORIUM, JONI RUTTER OF NCATS 6137 04:08:09,720 --> 04:08:13,360 ABOUT TO CLOSE THE MEETING AND 6138 04:08:13,360 --> 04:08:15,400 SEND US HOME, HAD BEEN TEXTING 6139 04:08:15,400 --> 04:08:16,400 WITH ME ALL DAY, SHE NOW THAT 6140 04:08:16,400 --> 04:08:19,440 WAS THE DATE ON WHICH THE FDA 6141 04:08:19,440 --> 04:08:23,640 WAS SUPPOSED TO ANNOUNCE A 6142 04:08:23,640 --> 04:08:24,000 DECISION. 6143 04:08:24,000 --> 04:08:24,840 NOTHING YET, JONI. 6144 04:08:24,840 --> 04:08:26,840 I GET THE CALL AT 4:30. 6145 04:08:26,840 --> 04:08:28,680 JONI'S UP THERE AT THE STAGE AT 6146 04:08:28,680 --> 04:08:29,640 THE PODIUM LIKE THIS. 6147 04:08:29,640 --> 04:08:31,280 ABOUT TO CLOSE THE MEETING. 6148 04:08:31,280 --> 04:08:37,400 I WALKED DOWN THE AISLE, 6149 04:08:37,400 --> 04:08:38,280 ATTRACTED HER ATTENTION, 6150 04:08:38,280 --> 04:08:38,800 TOUCHDOWN SALUTE. 6151 04:08:38,800 --> 04:08:40,000 RON, DO YOU HAVE ANY NEWS FOR 6152 04:08:40,000 --> 04:08:40,200 US? 6153 04:08:40,200 --> 04:08:42,120 CAN YOU STEP UP TO THE 6154 04:08:42,120 --> 04:08:43,600 MICROPHONE AND TELL US? 6155 04:08:43,600 --> 04:08:46,040 I WAS ABLE TO ANNOUNCE THAT WE 6156 04:08:46,040 --> 04:08:48,800 GOT OUR FIRST APPROVAL. 6157 04:08:48,800 --> 04:08:54,600 6158 04:08:54,600 --> 04:08:59,400 SO FROM THE FIRST TRIAL TO THE 6159 04:08:59,400 --> 04:09:01,640 APPROVAL, 8 YEARS VERSUS TYPICAL 6160 04:09:01,640 --> 04:09:03,680 10 TO 14 YEARS, AND WE'RE JUST 6161 04:09:03,680 --> 04:09:07,280 REALLY THRILLED TO HAVE OUR VERY 6162 04:09:07,280 --> 04:09:09,760 FIRST TREATMENT. 6163 04:09:09,760 --> 04:09:15,240 6164 04:09:15,240 --> 04:09:17,000 >> I THINK I HAVE THE HARDEST 6165 04:09:17,000 --> 04:09:18,120 JOB TODAY BECAUSE I'M SUPPOSED 6166 04:09:18,120 --> 04:09:19,880 TO MANAGE TIME WHEN THERE'S GOOD 6167 04:09:19,880 --> 04:09:22,480 NEWS TO BE SHARED. 6168 04:09:22,480 --> 04:09:25,320 I'M GOING TO TINTER OVER TO OUR 6169 04:09:25,320 --> 04:09:26,560 COLLEAGUE FROM THE ALS 6170 04:09:26,560 --> 04:09:27,720 COMMUNITY, I THINK I'M STARTING 6171 04:09:27,720 --> 04:09:28,280 WITH NEIL. 6172 04:09:28,280 --> 04:09:29,600 GOOD TO SEE YOU. 6173 04:09:29,600 --> 04:09:31,040 I'M GOING TO ENCOURAGE YOU TO 6174 04:09:31,040 --> 04:09:31,800 WATCH THE CLOCK. 6175 04:09:31,800 --> 04:09:33,440 WE'LL TRY AND KEEP YOU TO SIX 6176 04:09:33,440 --> 04:09:37,600 MINTS OR -- MINUTES TO HAVE TIME 6177 04:09:37,600 --> 04:09:40,080 FOR TIM AND QUESTIONS. 6178 04:09:40,080 --> 04:09:40,600 TAKE IT AWAY. 6179 04:09:40,600 --> 04:09:42,720 >> I'M SORRY I COULDN'T BE HERE 6180 04:09:42,720 --> 04:09:43,080 TODAY. 6181 04:09:43,080 --> 04:09:48,000 I'M ON COVID QUARANTINE. 6182 04:09:48,000 --> 04:09:49,560 I WAS LOOKING FORWARD TO MEETING 6183 04:09:49,560 --> 04:09:52,760 YOU AND BEING ON CAMPUS. 6184 04:09:52,760 --> 04:09:56,800 LIKE SOME CONDITIONS WE'VE 6185 04:09:56,800 --> 04:09:58,760 TALKED ABOUT, ALS IS NOT AS WELL 6186 04:09:58,760 --> 04:09:59,160 UNDERSTOOD. 6187 04:09:59,160 --> 04:10:02,160 THERE'S NOT A SINGLE GENE THAT'S 6188 04:10:02,160 --> 04:10:02,480 IMPLICATED. 6189 04:10:02,480 --> 04:10:04,440 SPECIFIC CAUSES ARE UNCLEAR. 6190 04:10:04,440 --> 04:10:05,760 THERE'S A LOT OF HETEROGENEITY 6191 04:10:05,760 --> 04:10:11,240 IN THE ILLNESS, NOT EASY TO LOOK 6192 04:10:11,240 --> 04:10:12,240 AT UNUSED NATURAL HISTORY 6193 04:10:12,240 --> 04:10:14,960 CONTROLS FOR EXAMPLE TO MAKE 6194 04:10:14,960 --> 04:10:16,160 INFERENCES ABOUT NEW DRUGS 6195 04:10:16,160 --> 04:10:18,520 BECAUSE OF THE HETEROGENEITY OF 6196 04:10:18,520 --> 04:10:23,680 THE ILLNESS. 6197 04:10:23,680 --> 04:10:24,760 SO NEXT SLIDE PLEASE. 6198 04:10:24,760 --> 04:10:26,160 WHEN WE TALK ABOUT SUCCESS IT'S 6199 04:10:26,160 --> 04:10:27,240 IMPORTANT TO CONSIDER WHAT IT 6200 04:10:27,240 --> 04:10:31,200 TAKES TO GET TO SUCCESS AND WHAT 6201 04:10:31,200 --> 04:10:35,440 WE ANTICIPATE IS THE HIGH RATE 6202 04:10:35,440 --> 04:10:38,640 OF FAILURE OF CLINICAL TRIALS OF 6203 04:10:38,640 --> 04:10:39,240 DRUG CANDIDATES. 6204 04:10:39,240 --> 04:10:41,040 PART OF OUR STRATEGY IS TO 6205 04:10:41,040 --> 04:10:44,000 FOCUS, TO PLAN FOR THAT, AND TO 6206 04:10:44,000 --> 04:10:45,880 HAVE A DIVERSIFIED PORTFOLIO SO 6207 04:10:45,880 --> 04:10:47,920 WE CAN GET SUCCESSES. 6208 04:10:47,920 --> 04:10:50,760 AND THAT STRATEGY HAS BEEN 6209 04:10:50,760 --> 04:10:51,880 FAIRLY SUCCESSFUL. 6210 04:10:51,880 --> 04:10:54,520 AND SO THE KEY ASPECT OF HAVING 6211 04:10:54,520 --> 04:10:57,480 THIS STRATEGY IS TO FOCUS ON 6212 04:10:57,480 --> 04:10:57,720 TIME. 6213 04:10:57,720 --> 04:10:58,920 TIME TO POTENTIAL IMPACT. 6214 04:10:58,920 --> 04:10:59,880 BECAUSE WHEN YOU'RE DEALING WITH 6215 04:10:59,880 --> 04:11:02,360 A DISEASE WHERE THERE ARE A LOT 6216 04:11:02,360 --> 04:11:05,440 OF UNKNOWNS, IT'S VERY LOGICAL 6217 04:11:05,440 --> 04:11:06,440 AND CERTAINLY CAREFUL SCIENTISTS 6218 04:11:06,440 --> 04:11:08,200 WANT TO SAY LET'S FOCUS REALLY 6219 04:11:08,200 --> 04:11:11,120 CAREFULLY ON THE BASIC SCIENCE 6220 04:11:11,120 --> 04:11:13,320 OF FUNDAMENTALS, THE BIOLOGY, 6221 04:11:13,320 --> 04:11:15,720 GENETICS, POTENTIAL CAUSES AND 6222 04:11:15,720 --> 04:11:16,920 RISKS OF DISEASE, SOME WORK CAN 6223 04:11:16,920 --> 04:11:19,040 TAKE A VERY LONG TIME TO HAPPEN. 6224 04:11:19,040 --> 04:11:21,000 SO WE DO NEED TO DO THAT WORK 6225 04:11:21,000 --> 04:11:23,440 BUT WHILE IT'S HAPPENING IN 6226 04:11:23,440 --> 04:11:25,240 PARALLEL, WE NEED TO DO MORE 6227 04:11:25,240 --> 04:11:27,320 ADVANCED WORK, MORE CLINICAL 6228 04:11:27,320 --> 04:11:29,400 WORK, AT THE SAME TIME, ACCEPT 6229 04:11:29,400 --> 04:11:31,600 HIGHER RATE OF FAILURE IN 6230 04:11:31,600 --> 04:11:33,960 CLINICAL TRIALS AND CLINICAL 6231 04:11:33,960 --> 04:11:34,440 RESEARCH. 6232 04:11:34,440 --> 04:11:36,760 WE'VE BEEN DELIBERATE AND 6233 04:11:36,760 --> 04:11:38,160 FOCUSING ON DIVERSE CANDIDATES, 6234 04:11:38,160 --> 04:11:39,800 BRINGING NEW SCIENTISTS AND NEW 6235 04:11:39,800 --> 04:11:41,640 SCIENTIFIC TEAMS INTO THE FIELD, 6236 04:11:41,640 --> 04:11:42,640 AND WORKING ON OUR 6237 04:11:42,640 --> 04:11:45,840 INFRASTRUCTURE TO MAKE SURE WE 6238 04:11:45,840 --> 04:11:48,640 HAVE THE SPECIMENS, RESOURCES, 6239 04:11:48,640 --> 04:11:49,760 THE NETWORKS OF CLINICAL 6240 04:11:49,760 --> 04:11:51,920 TRIALISTS TO GET TRIALS MOVING 6241 04:11:51,920 --> 04:11:52,600 QUICKLY. 6242 04:11:52,600 --> 04:11:54,680 NOW WE HAVE DOZENS OF COMPOUNDS 6243 04:11:54,680 --> 04:11:58,400 UNDER STUDY IN HUMANS, WE SEE 6244 04:11:58,400 --> 04:12:00,760 FOLLOWING FROM NIH AND OTHER 6245 04:12:00,760 --> 04:12:03,880 SOURCES, $3 FOR EVERY DOLLAR WE 6246 04:12:03,880 --> 04:12:06,280 INVEST IN DRUG PORTFOLIO, IT'S 6247 04:12:06,280 --> 04:12:08,160 $6 OF FOLLOW-ON FUNDING, HUGE 6248 04:12:08,160 --> 04:12:09,440 INCREASE IN FEDERAL ALS RESEARCH 6249 04:12:09,440 --> 04:12:12,400 FUNDING IN PART BECAUSE OF 6250 04:12:12,400 --> 04:12:15,040 WHAT'S GOING ON AT NINDS AND 6251 04:12:15,040 --> 04:12:17,840 DEMENTIA DISCOVERY FUNDS, BUT 6252 04:12:17,840 --> 04:12:22,280 ALSO THROUGH ADVOCACY WORK 6253 04:12:22,280 --> 04:12:25,240 THROUGH DEPARTMENT OF DEFENSE 6254 04:12:25,240 --> 04:12:27,880 MORE THAN 300% INCREASE THROUGH 6255 04:12:27,880 --> 04:12:29,080 2013, THE ICE BUCKET CHALLENGES 6256 04:12:29,080 --> 04:12:33,120 MADE A HUGE IMPACT, MORE MONEY 6257 04:12:33,120 --> 04:12:33,720 THROUGH PHILANTHROPIC SPACE, 6258 04:12:33,720 --> 04:12:35,320 MORE FUNDING IN THE FEDERAL 6259 04:12:35,320 --> 04:12:36,520 SPACE. 6260 04:12:36,520 --> 04:12:36,960 NEXT PAGE. 6261 04:12:36,960 --> 04:12:40,760 THE NEXT PART IS HOW WE WORK 6262 04:12:40,760 --> 04:12:41,680 WITH THE FDA. 6263 04:12:41,680 --> 04:12:44,960 WE'VE GONE THROUGH THE FDA 6264 04:12:44,960 --> 04:12:47,240 PROCESS, WE'VE HAD THE 6265 04:12:47,240 --> 04:12:48,440 PATIENT-FOCUSED DRUG DEVELOPMENT 6266 04:12:48,440 --> 04:12:49,560 MEETINGS WITH THE FDA. 6267 04:12:49,560 --> 04:12:53,360 WE WORKED ON A TRIAL GUIDANCE 6268 04:12:53,360 --> 04:12:57,520 THAT WAS ADOPTED, BECAME 6269 04:12:57,520 --> 04:12:58,520 OFFICIAL, IN 2019. 6270 04:12:58,520 --> 04:13:00,040 I THINK WHAT'S EVEN MORE 6271 04:13:00,040 --> 04:13:02,560 IMPORTANT IS WHEN A DRUG DOES GO 6272 04:13:02,560 --> 04:13:05,080 TO FDA FOR APPROVAL ON OCCASION 6273 04:13:05,080 --> 04:13:06,160 THE ASSOCIATION DOES TAKE A 6274 04:13:06,160 --> 04:13:08,840 STAND IN THAT PROCESS. 6275 04:13:08,840 --> 04:13:11,640 WE MAKE STATEMENTS, SOME CASES 6276 04:13:11,640 --> 04:13:14,080 ENGAGE IN ADVOCACY OUTSIDE OF 6277 04:13:14,080 --> 04:13:15,240 THIS PDUFA PROCESS, TRIAL 6278 04:13:15,240 --> 04:13:15,840 GUIDANCE. 6279 04:13:15,840 --> 04:13:17,600 AND WE HAVE TO BE CAREFUL ABOUT 6280 04:13:17,600 --> 04:13:19,640 OUR CONFLICTS OF INTEREST. 6281 04:13:19,640 --> 04:13:23,440 WE HAVE PHARMA PARTNERS WHO 6282 04:13:23,440 --> 04:13:25,040 SPONSOR ACTIVITIES, WE FUNDED 6283 04:13:25,040 --> 04:13:28,080 SOME DRUGS UNDER REVIEW BY THE 6284 04:13:28,080 --> 04:13:28,280 FDA. 6285 04:13:28,280 --> 04:13:29,480 SOMETIMES IT'S BOTH. 6286 04:13:29,480 --> 04:13:32,760 ON TOP OF THAT, WE ALL AS STAFF 6287 04:13:32,760 --> 04:13:34,080 KNOW PEOPLE WHO ARE 6288 04:13:34,080 --> 04:13:36,720 PARTICIPATING IN THESE PHASE 3 6289 04:13:36,720 --> 04:13:37,000 TRIALS. 6290 04:13:37,000 --> 04:13:37,840 WE RESPECT THEM. 6291 04:13:37,840 --> 04:13:38,720 THEY SHAPE OUR OPINIONS. 6292 04:13:38,720 --> 04:13:39,760 THAT'S WHY WE WORK REALLY HARD 6293 04:13:39,760 --> 04:13:43,960 TO MAKE SURE WE HAVE AN 6294 04:13:43,960 --> 04:13:44,800 INDEPENDENT OUTSIDE REVIEW OF 6295 04:13:44,800 --> 04:13:45,920 DRUG DATA BEFORE WE MAKE 6296 04:13:45,920 --> 04:13:47,120 STATEMENTS ON WHETHER WE THINK 6297 04:13:47,120 --> 04:13:48,160 THE FDA SHOULD APPROVE SOMETHING 6298 04:13:48,160 --> 04:13:48,840 OR NOT. 6299 04:13:48,840 --> 04:13:51,160 WE CAN ONLY DO THAT IF THE 6300 04:13:51,160 --> 04:13:53,560 COMPANY IS WILLING TO LET US 6301 04:13:53,560 --> 04:13:55,760 HAVE ACCESS TO THAT DATA BECAUSE 6302 04:13:55,760 --> 04:14:01,880 SOME IS PRY PRY TEAR -- 6303 04:14:01,880 --> 04:14:02,320 PROPRIETARY. 6304 04:14:02,320 --> 04:14:04,440 WE WOULD LIKE THE FDA TO VIEW 6305 04:14:04,440 --> 04:14:06,400 DRUG APPROVALS IN THE ALS SPACE 6306 04:14:06,400 --> 04:14:09,600 WITHIN THE CONTEXT OF ALS. 6307 04:14:09,600 --> 04:14:12,640 RARE DISEASE WITH NO CURE THAT'S 6308 04:14:12,640 --> 04:14:14,520 RAPIDLY PROGRESSIVE SO THAT'S A 6309 04:14:14,520 --> 04:14:17,240 DIFFERENT STANDARD THAT ONE 6310 04:14:17,240 --> 04:14:20,960 WOULD EXPECT FOR A DISEASE THAT 6311 04:14:20,960 --> 04:14:22,480 HAS A CURE. 6312 04:14:22,480 --> 04:14:24,600 STAND AROUND FOR HEARTBURN 6313 04:14:24,600 --> 04:14:26,000 MEDICATION, ALS MEDICATION, THEY 6314 04:14:26,000 --> 04:14:27,000 SHOULD BE DIFFERENT. 6315 04:14:27,000 --> 04:14:29,640 NEXT SLIDE, WE HAD AN 6316 04:14:29,640 --> 04:14:33,680 INTERESTING STUDY FOR APPROVAL 6317 04:14:33,680 --> 04:14:40,480 OF A DRUG, AMX 0035, APPROVED 6318 04:14:40,480 --> 04:14:41,880 UNDER THE NAME RELIVRIO. 6319 04:14:41,880 --> 04:14:45,200 THE ADVISORY COMMITTEE IN MARCH 6320 04:14:45,200 --> 04:14:48,360 OF 2022 WAS ASKED TO ESTABLISH 6321 04:14:48,360 --> 04:14:50,360 CONCLUSION IT IS EFFECTIVE IN 6322 04:14:50,360 --> 04:14:52,520 TREATMENT OF PATIENTS WITH ALS, 6323 04:14:52,520 --> 04:14:54,080 TOOK A VOTE, THOUGHT THERE 6324 04:14:54,080 --> 04:14:57,360 WASN'T ENOUGH EVIDENCE TO 6325 04:14:57,360 --> 04:14:58,400 ESTABLISH CONCLUSION. 6326 04:14:58,400 --> 04:15:01,400 SO WE PUSHED BACK, THE COMMUNITY 6327 04:15:01,400 --> 04:15:04,440 PUSHED BACK, THERE WAS 6328 04:15:04,440 --> 04:15:06,640 PETITIONS, AND THEY HELD A 6329 04:15:06,640 --> 04:15:07,840 SECOND ADVISORY COMMITTEE AND 6330 04:15:07,840 --> 04:15:09,840 ASKED THE SAME DRUG, ASKED A 6331 04:15:09,840 --> 04:15:10,960 DIFFERENT QUESTION. 6332 04:15:10,960 --> 04:15:13,080 THAT IS, IS THE AVAILABLE 6333 04:15:13,080 --> 04:15:15,760 EVIDENCE OF EFFECTIVENESS 6334 04:15:15,760 --> 04:15:18,080 SUFFICIENT TO SUPPORT APPROVAL? 6335 04:15:18,080 --> 04:15:20,120 DIFFERENT STANDARD. 6336 04:15:20,120 --> 04:15:22,640 YOU MAY TAKE INTO ACCOUNT UNMET 6337 04:15:22,640 --> 04:15:25,960 NEED AND STATUS OF TRIAL AND SO 6338 04:15:25,960 --> 04:15:27,280 ON AND SERIOUSNESS. 6339 04:15:27,280 --> 04:15:30,400 A PUBLIC HEALTH APPROACH, ASKING 6340 04:15:30,400 --> 04:15:32,320 PEOPLE TO CONSIDER CONTEXT OF 6341 04:15:32,320 --> 04:15:34,800 DRUG, DISEASE, WHEN MAKING 6342 04:15:34,800 --> 04:15:35,080 DECISION. 6343 04:15:35,080 --> 04:15:37,160 THAT ADVISORY COMMITTEE DID VOTE 6344 04:15:37,160 --> 04:15:37,680 FOR APPROVAL. 6345 04:15:37,680 --> 04:15:40,560 SO IF YOU GO TO THE NEXT SLIDE 6346 04:15:40,560 --> 04:15:40,920 PLEASE. 6347 04:15:40,920 --> 04:15:42,680 SO, WHAT'S THE SUCCESS OF THIS? 6348 04:15:42,680 --> 04:15:45,880 WE'VE GONE FOR A LONG TIME WITH 6349 04:15:45,880 --> 04:15:49,600 SLOW CHANGES IN THE TREATMENT 6350 04:15:49,600 --> 04:15:51,680 LANDSCAPE, AND GRADUAL INCREASE 6351 04:15:51,680 --> 04:15:55,640 IN ACCELERATION, IN APPROVAL OF 6352 04:15:55,640 --> 04:15:58,360 TREATMENTS, IN THE PAST YEAR TWO 6353 04:15:58,360 --> 04:15:59,440 TREATMENTS APPROVED, VERY 6354 04:15:59,440 --> 04:16:00,760 UNUSUAL IN THE ALS SPACE BECAUSE 6355 04:16:00,760 --> 04:16:07,800 APPROVAL PRIOR TO THAT WAS IN 6356 04:16:07,800 --> 04:16:08,320 2017. 6357 04:16:08,320 --> 04:16:11,480 ARE WE READY FOR THIS CHANGE IN 6358 04:16:11,480 --> 04:16:12,800 ALS TREATMENT? 6359 04:16:12,800 --> 04:16:14,880 WE'RE TALKING ABOUT NOW MULTIPLE 6360 04:16:14,880 --> 04:16:16,760 TREATMENTS THAT ALL HAVE MODEST 6361 04:16:16,760 --> 04:16:18,240 EFFECTS, SO PERHAPS THEY CAN 6362 04:16:18,240 --> 04:16:20,120 WORK IN COMBINATION, PERHAPS 6363 04:16:20,120 --> 04:16:21,440 THEY CAN'T. 6364 04:16:21,440 --> 04:16:22,200 WE'RE HAVING CHALLENGES SINCE 6365 04:16:22,200 --> 04:16:24,920 THEY ARE EXPENSIVE MAKING SURE 6366 04:16:24,920 --> 04:16:26,320 EVERYONE WHO CAN BENEFIT CAN GET 6367 04:16:26,320 --> 04:16:27,440 THESE TREATMENTS. 6368 04:16:27,440 --> 04:16:29,320 WE HAVE TROUBLE GETTING PEOPLE 6369 04:16:29,320 --> 04:16:31,720 DIAGNOSED AND REFERRED TO 6370 04:16:31,720 --> 04:16:32,600 SPECIALTY CENTERS. 6371 04:16:32,600 --> 04:16:35,240 AND THEN PEOPLE STILL HAVE 6372 04:16:35,240 --> 04:16:38,520 COMPLICATIONS FROM ALS CAUSING 6373 04:16:38,520 --> 04:16:45,080 SIGNIFICANT HARM, FALLS, ET 6374 04:16:45,080 --> 04:16:45,520 CETERA. 6375 04:16:45,520 --> 04:16:46,600 INSURANCE COMPANIES, WE DON'T 6376 04:16:46,600 --> 04:16:47,560 NECESSARILY HAVE CLINICAL BASE 6377 04:16:47,560 --> 04:16:48,880 FOR HOW MUCH THINGS COST TO MAKE 6378 04:16:48,880 --> 04:16:51,880 SURE WE KNOW HOW TO DELIVER CARE 6379 04:16:51,880 --> 04:16:52,200 EFFECTIVELY. 6380 04:16:52,200 --> 04:16:54,360 SO THIS IS A CHALLENGE. 6381 04:16:54,360 --> 04:16:56,000 AND CANCER HAS BEEN DEALING WITH 6382 04:16:56,000 --> 04:16:58,120 ISSUES OF HOW TO GET PEOPLE 6383 04:16:58,120 --> 04:17:00,360 DIAGNOSED AND GET PEOPLE IN 6384 04:17:00,360 --> 04:17:01,200 TREATMENT, SO ACTUAL SURVIVAL 6385 04:17:01,200 --> 04:17:04,320 CAN IMPROVE FOR A LONG TIME. 6386 04:17:04,320 --> 04:17:07,080 WHEN I TALKED ABOUT THIS WITH MY 6387 04:17:07,080 --> 04:17:10,240 RESEARCH COLLEAGUES I DON'T GET 6388 04:17:10,240 --> 04:17:12,320 THE SAME EXCITEMENT OR ATTENTION 6389 04:17:12,320 --> 04:17:15,720 TO FIGURING OUT HOW TO IMPROVE 6390 04:17:15,720 --> 04:17:17,360 CARE. 6391 04:17:17,360 --> 04:17:21,640 PART MIGHT BE THE WAY WE'RE 6392 04:17:21,640 --> 04:17:22,720 ALLOCATING FUNDING. 6393 04:17:22,720 --> 04:17:25,680 NCI SPENDS 25% ON TREATING 6394 04:17:25,680 --> 04:17:27,240 CANCER AND IMPROVING 6395 04:17:27,240 --> 04:17:27,680 SURVIVORSHIP. 6396 04:17:27,680 --> 04:17:30,600 THE EXTRAMURAL BUDGET AT NINDS 6397 04:17:30,600 --> 04:17:32,920 IS ONLY 4% FOR CLINICAL 6398 04:17:32,920 --> 04:17:34,200 RESEARCH, WHICH ISN'T EVEN QUITE 6399 04:17:34,200 --> 04:17:34,760 THE SAME THING. 6400 04:17:34,760 --> 04:17:36,320 I THINK WE HAVE SOME WORK TO DO 6401 04:17:36,320 --> 04:17:38,760 BOTH ON THE FEDERAL SIDE AND 6402 04:17:38,760 --> 04:17:40,480 CERTAINLY AT THE ASSOCIATION 6403 04:17:40,480 --> 04:17:43,560 SIDE, INCLUDING OUR CAPACITY TO 6404 04:17:43,560 --> 04:17:44,640 STUDY QUALITY OF CARE, 6405 04:17:44,640 --> 04:17:45,600 INCREASING NUMBER OF SCIENTISTS 6406 04:17:45,600 --> 04:17:47,480 TRAINED TO DO THIS KIND OF WORK, 6407 04:17:47,480 --> 04:17:50,240 WE LAUNCHED A QUALITY OF CARE 6408 04:17:50,240 --> 04:17:51,640 RFA AT THE ASSOCIATION THIS YEAR 6409 04:17:51,640 --> 04:17:53,160 AND THIS IS THE FIRST TIME WE'VE 6410 04:17:53,160 --> 04:17:53,840 DONE THAT. 6411 04:17:53,840 --> 04:17:57,000 I THINK THE WHOLE FIELD HAS TO 6412 04:17:57,000 --> 04:18:00,080 ACCOMMODATE TO THE IDEA THAT NEW 6413 04:18:00,080 --> 04:18:01,480 TREATMENTS ARE HERE AND HAVE TO 6414 04:18:01,480 --> 04:18:03,040 FIGURE OUT HOW TO MAKE THEM 6415 04:18:03,040 --> 04:18:03,480 WORK. 6416 04:18:03,480 --> 04:18:04,720 I BELIEVE THAT'S MY LAST SLIDE. 6417 04:18:04,720 --> 04:18:07,960 THANK YOU. 6418 04:18:07,960 --> 04:18:13,200 6419 04:18:13,200 --> 04:18:15,000 6420 04:18:15,000 --> 04:18:16,720 >> IT'S INTERESTING WHEN YOU 6421 04:18:16,720 --> 04:18:17,600 THINK ABOUT THE ACHIEVEMENT, 6422 04:18:17,600 --> 04:18:19,120 HAVING THERAPIES BUT THEN HOW DO 6423 04:18:19,120 --> 04:18:21,760 YOU HELP PATIENTS AND FAMILIES 6424 04:18:21,760 --> 04:18:24,840 NAVIGATE THAT LANDSCAPE, WHAT 6425 04:18:24,840 --> 04:18:25,960 DOES ACCESS LOOK LIKE, A LOT TO 6426 04:18:25,960 --> 04:18:27,440 BE CONSIDERED. 6427 04:18:27,440 --> 04:18:28,760 THERE ARE QUESTIONS COMING IN SO 6428 04:18:28,760 --> 04:18:30,400 WHAT STANDS BETWEEN US AND 6429 04:18:30,400 --> 04:18:31,840 QUESTIONS IS DR. TIM MILLER. 6430 04:18:31,840 --> 04:18:34,040 WE'RE GOING TO SHOW TIM'S 6431 04:18:34,040 --> 04:18:35,240 PRESENTATION AND OPEN FOR SOME 6432 04:18:35,240 --> 04:18:37,640 CONVERSATION. 6433 04:18:37,640 --> 04:18:43,640 6434 04:18:43,640 --> 04:18:48,560 6435 04:18:48,560 --> 04:18:49,880 >> I'M TIM MILLER, A NEUROLOGY 6436 04:18:49,880 --> 04:18:52,960 AT WASHINGTON UNIVERSITY IN 6437 04:18:52,960 --> 04:18:53,280 ST. LOUIS. 6438 04:18:53,280 --> 04:18:55,600 I'M DELIGHTED TO PARTICIPATE IN 6439 04:18:55,600 --> 04:18:58,320 THIS NON-PROFIT FORUM. 6440 04:18:58,320 --> 04:19:04,880 I'LL START WITH INDUSTRY 6441 04:19:04,880 --> 04:19:06,240 COLLABORATIONS, IONIS 6442 04:19:06,240 --> 04:19:09,520 PHARMACEUTICALS, AND BIOGEN, 6443 04:19:09,520 --> 04:19:11,040 RUNNING CLINICAL STUDIES. 6444 04:19:11,040 --> 04:19:12,920 SUCCESS STORIES IN ALS, 6445 04:19:12,920 --> 04:19:17,000 HIGHLIGHTING TWO IN FIVE TO FIX 6446 04:19:17,000 --> 04:19:20,160 MINUTES, THERAPY HAS BEEN SLOW 6447 04:19:20,160 --> 04:19:23,200 AND IMPROVES, AND DEMONSTRATION 6448 04:19:23,200 --> 04:19:26,520 OF USEFUL BLOOD BIOMARKER. 6449 04:19:26,520 --> 04:19:30,440 TWO OTHERS, FASTER WAY TO TEST 6450 04:19:30,440 --> 04:19:34,480 NEW THERAPIES AND EDUCATE THE 6451 04:19:34,480 --> 04:19:34,840 COMMUNITY. 6452 04:19:34,840 --> 04:19:39,200 90% IS SPORADIC, 10% IS 6453 04:19:39,200 --> 04:19:39,960 FAMILIAL. 6454 04:19:39,960 --> 04:19:44,480 MUTATIONS IN GENE SUPEROXIDE 6455 04:19:44,480 --> 04:19:49,480 DISMUTASE 1 ACCOUNTS FOR 10 TO 6456 04:19:49,480 --> 04:19:51,040 20%, THAT'S 300 TO 350 PEOPLE IN 6457 04:19:51,040 --> 04:19:52,760 THE UNITED STATES. 6458 04:19:52,760 --> 04:19:56,960 THE RATIONALE FOR LOWERING SOD1 6459 04:19:56,960 --> 04:19:59,240 IS MUTANT SOD1 ACQUIRES NEW 6460 04:19:59,240 --> 04:20:01,440 TOXICS PROPERTY, DECREASING IS 6461 04:20:01,440 --> 04:20:02,960 LIKELY TO BE BENEFICIAL, 6462 04:20:02,960 --> 04:20:07,440 DECREASING IS LIKELY TO BE SAFE 6463 04:20:07,440 --> 04:20:10,080 MUTATIONS IN THE GENES CAUSE 6464 04:20:10,080 --> 04:20:17,440 ABNORMAL RNA, THAT LEADS TO THE 6465 04:20:17,440 --> 04:20:20,080 TOXIC PROTEIN, ANTISENSE DRUGS 6466 04:20:20,080 --> 04:20:22,560 CAN BLOCK DISEASE CAUSING RNA 6467 04:20:22,560 --> 04:20:23,560 AND TOXIC PROTEIN. 6468 04:20:23,560 --> 04:20:30,000 TALKING ABOUT THE VALOR TRIAL, 6469 04:20:30,000 --> 04:20:33,600 PHASE 3 USING TOFERSEN, 6470 04:20:33,600 --> 04:20:36,400 ANTISENSE OLIGONUCLEOTIDE, 72 6471 04:20:36,400 --> 04:20:38,080 PARTICIPANTS ON DRUGS, 36 ON 6472 04:20:38,080 --> 04:20:39,760 PLACEBO, THREE DOSES IN THE 6473 04:20:39,760 --> 04:20:44,160 FIRST MONTH, A DOSE ABOUT ONCE A 6474 04:20:44,160 --> 04:20:45,240 MONTH AFTER THAT. 6475 04:20:45,240 --> 04:20:46,600 IT DOES NOT CROSS THE 6476 04:20:46,600 --> 04:20:47,680 BLOOD-BRAIN BARRIER, DELIVERED 6477 04:20:47,680 --> 04:20:51,280 TO THE BRAIN AND SPINAL CORD 6478 04:20:51,280 --> 04:20:53,680 INTRATHECAL SO THE AREA BELOW 6479 04:20:53,680 --> 04:20:58,280 THE SPINAL CORD, INTRODUCING THE 6480 04:20:58,280 --> 04:21:01,000 DRUG, THEN THROUGHOUT THE BRAIN 6481 04:21:01,000 --> 04:21:03,960 AND SPINAL CORD. 6482 04:21:03,960 --> 04:21:06,040 WHEN INTRODUCING, THE GROUP IN 6483 04:21:06,040 --> 04:21:08,560 BLUE ARE ON PLACEBO UNTIL HERE 6484 04:21:08,560 --> 04:21:11,040 AT 28 DAYS, OPEN LABEL EXTENSION 6485 04:21:11,040 --> 04:21:11,840 THEY RECEIVE DRUG. 6486 04:21:11,840 --> 04:21:13,480 GROUP IN RED ON THE DRUG FROM 6487 04:21:13,480 --> 04:21:14,160 THE BEGINNING. 6488 04:21:14,160 --> 04:21:18,520 BOTH GROUPS YOU SEE LOWERING IN 6489 04:21:18,520 --> 04:21:19,480 THE CEREBROSPINAL FLUID, EXACTLY 6490 04:21:19,480 --> 04:21:21,800 WHAT THE DRUG IS SUPPOSED TO DO. 6491 04:21:21,800 --> 04:21:24,720 TO READ OUT WHETHER IT HAD 6492 04:21:24,720 --> 04:21:27,360 EFFECT WE LOOKED AT NEURO 6493 04:21:27,360 --> 04:21:28,600 FILAMENTS, PROTEINS THAT ARE IN 6494 04:21:28,600 --> 04:21:30,480 THE AXON, CONNECTION BETWEEN THE 6495 04:21:30,480 --> 04:21:32,760 MOTOR NEURON AND OUT HERE, THE 6496 04:21:32,760 --> 04:21:33,680 MUSCLE. 6497 04:21:33,680 --> 04:21:35,720 CONNECTING IN BETWEEN THE AXON 6498 04:21:35,720 --> 04:21:37,800 WHEN THOSE AXONS ARE INJURED AS 6499 04:21:37,800 --> 04:21:43,200 OCCURS IN ALS, OTHER DISORDERS, 6500 04:21:43,200 --> 04:21:50,080 THEY LEAK OUT WHERE WE CAN 6501 04:21:50,080 --> 04:21:54,000 MEASURE THEM. 6502 04:21:54,000 --> 04:21:56,200 THEY PREDICT SURVIVAL, AT FOUR 6503 04:21:56,200 --> 04:21:59,080 YEARS WITH LOW NEUROFILAMENT 6504 04:21:59,080 --> 04:22:01,800 MORE THAN 60% SURVIVING. 6505 04:22:01,800 --> 04:22:04,440 NEUROFILAMENTS WERE LOWERED IN 6506 04:22:04,440 --> 04:22:06,960 THIS GROUP HERE, AND THEN ON 6507 04:22:06,960 --> 04:22:10,240 DRUG NEUROFILAMENTS COME DOWN. 6508 04:22:10,240 --> 04:22:11,200 MY INTERPRETATION IS SUBSTANTIAL 6509 04:22:11,200 --> 04:22:15,880 SLOWING OF THE DISEASE PROCESS. 6510 04:22:15,880 --> 04:22:19,640 THIS DID NOT MEET A PRIMARY 6511 04:22:19,640 --> 04:22:20,840 ENDPOINT, ALS FUNCTIONAL RATINGS 6512 04:22:20,840 --> 04:22:23,160 SCALE NOT MUCH DIFFERENT AT 28 6513 04:22:23,160 --> 04:22:23,360 WEEKS. 6514 04:22:23,360 --> 04:22:30,080 OPEN LABEL EXTENSION, 52 WEEKS, 6515 04:22:30,080 --> 04:22:30,360 , 6516 04:22:30,360 --> 04:22:31,480 YOU SEE A DIFFERENCE BETWEEN 6517 04:22:31,480 --> 04:22:32,440 DRUGS AND PLACEBO. 6518 04:22:32,440 --> 04:22:34,680 THIS IS MY FAVORITE PIECE OF 6519 04:22:34,680 --> 04:22:38,040 DATA FROM THIS STUDY. 6520 04:22:38,040 --> 04:22:48,600 THIS IS A HAND-HELD DYNAMOMETRY, 6521 04:22:53,600 --> 04:22:55,360 INCREASE IN STRENGTH, 27% OF THE 6522 04:22:55,360 --> 04:23:02,480 PEOPLE INITIALLY ON THE DRUG HAD 6523 04:23:02,480 --> 04:23:05,200 INCREASE IN STRENGTH. 6524 04:23:05,200 --> 04:23:07,960 I ASKED THE GROUP HERE, 178 6525 04:23:07,960 --> 04:23:11,360 YEARS OF TAKING CARE OF 6526 04:23:11,360 --> 04:23:12,960 PATIENTS, THE ANSWER IS ZERO. 6527 04:23:12,960 --> 04:23:16,720 WE HAVEN'T SEEN SOMEONE IN OUR 6528 04:23:16,720 --> 04:23:18,160 CENTER GETTING BETTER. 6529 04:23:18,160 --> 04:23:21,760 IN THIS STUDY 27% INCREASED 6530 04:23:21,760 --> 04:23:22,000 STRENGTH. 6531 04:23:22,000 --> 04:23:22,720 REMARKABLE STORIES, ONE STORY 6532 04:23:22,720 --> 04:23:26,360 FROM THE FDA ADVISORY COMMITTEE, 6533 04:23:26,360 --> 04:23:30,960 I'M DOING THINGS I COULD NOT DO, 6534 04:23:30,960 --> 04:23:34,240 LIMP HAS GONE FROM OBVIOUS TO 6535 04:23:34,240 --> 04:23:36,000 BARELY PER SUSCEPTIBLE, I CAN GO 6536 04:23:36,000 --> 04:23:37,760 TO MY OFFICE AGAIN, SHOWER 6537 04:23:37,760 --> 04:23:41,240 WITHOUT MY SHOWER CHAIR. 6538 04:23:41,240 --> 04:23:43,560 CONCLUSIONS LOWERING SOD1 LOWERS 6539 04:23:43,560 --> 04:23:45,200 NEURO FILAMENT, SUBSTANTIAL 6540 04:23:45,200 --> 04:23:47,360 SLOWING OF THE NEURODEGENERATIVE 6541 04:23:47,360 --> 04:23:49,800 DISEASE PROSET. 6542 04:23:49,800 --> 04:23:51,000 STABILIZATION OF FUNCTION, 6543 04:23:51,000 --> 04:23:52,400 BREATHING, STRENGTH, SOME 6544 04:23:52,400 --> 04:23:53,200 EVIDENCE OF IMPROVING, 6545 04:23:53,200 --> 04:23:55,400 REMARKABLE INDIVIDUAL STORIES. 6546 04:23:55,400 --> 04:24:00,960 OVERALL WELL TOLERATED, THERE 6547 04:24:00,960 --> 04:24:03,480 WERE NOTEWORTHY SIDE EFFECTS. 6548 04:24:03,480 --> 04:24:06,080 I'LL BE HAPPY TO DISCUSS. 6549 04:24:06,080 --> 04:24:09,040 THIS WAS APPROVED BY FDA APRIL 6550 04:24:09,040 --> 04:24:10,560 THIS YEAR. 6551 04:24:10,560 --> 04:24:13,880 ALS IS A TREATABLE DISORDER, THE 6552 04:24:13,880 --> 04:24:18,040 MOST PRO FOUND CONCLUSION, YOU 6553 04:24:18,040 --> 04:24:25,440 NEED THE RIGHT DRUG, 6554 04:24:25,440 --> 04:24:28,520 NEUROFILAMENT IS ESTABLISHED AS 6555 04:24:28,520 --> 04:24:30,200 A THERAPY-RESPONSE IMBIOMARKER. 6556 04:24:30,200 --> 04:24:32,680 IN MY OPINION PREDICTIVE OF 6557 04:24:32,680 --> 04:24:34,080 SUCCESS OR FAILURE, EFFECTIVE 6558 04:24:34,080 --> 04:24:35,440 DRUG MAY NOT CHANGE 6559 04:24:35,440 --> 04:24:35,800 NEUROFILAMENT. 6560 04:24:35,800 --> 04:24:38,600 I WANT TO GIVE A SHOUT OUT TO 6561 04:24:38,600 --> 04:24:39,920 THE STUDY PARTICIPANTS, WITHOUT 6562 04:24:39,920 --> 04:24:41,240 THEM THIS STUDY OF COURSE WOULD 6563 04:24:41,240 --> 04:24:42,080 NOT GO. 6564 04:24:42,080 --> 04:24:44,520 ALSO TO PRINCIPAL INVESTIGATORS 6565 04:24:44,520 --> 04:24:47,120 AND HARD-WORKING SITE STAFF, 6566 04:24:47,120 --> 04:24:51,520 STEERING COMMITTEE AND MY 6567 04:24:51,520 --> 04:24:52,320 COLLEAGUES AT BIOGEN. 6568 04:24:52,320 --> 04:24:55,280 THANK YOU AND I LOOK FORWARD TO 6569 04:24:55,280 --> 04:24:55,560 QUESTIONS. 6570 04:24:55,560 --> 04:24:58,080 [APPLAUSE] 6571 04:24:58,080 --> 04:25:04,120 6572 04:25:04,120 --> 04:25:05,040 6573 04:25:05,040 --> 04:25:07,000 HE MAY BE JOINING US. 6574 04:25:07,000 --> 04:25:08,880 AH, THERE HE IS. 6575 04:25:08,880 --> 04:25:09,720 >> I'M HERE. 6576 04:25:09,720 --> 04:25:11,600 GREAT TO BE HERE. 6577 04:25:11,600 --> 04:25:12,080 REALLY ENJOYED THE DAY. 6578 04:25:12,080 --> 04:25:15,920 >> THANK YOU SO MUCH. 6579 04:25:15,920 --> 04:25:17,160 WE'RE RUNNING BEHIND BUT WE'LL 6580 04:25:17,160 --> 04:25:19,680 TAKE THE EXTRA TEN MINUTES FOR 6581 04:25:19,680 --> 04:25:20,000 QUESTIONS. 6582 04:25:20,000 --> 04:25:22,120 I HAVE QUESTIONS THAT WE THOUGHT 6583 04:25:22,120 --> 04:25:23,760 ABOUT BUT KNOWING THAT WE ONLY 6584 04:25:23,760 --> 04:25:24,880 HAVE TEN MINUTES LEFT I WANT TO 6585 04:25:24,880 --> 04:25:28,000 START WITH QUESTIONS FROM THE 6586 04:25:28,000 --> 04:25:32,000 AUDIENCE IF ANYONE HAS ANY 6587 04:25:32,000 --> 04:25:34,120 QUESTIONS. 6588 04:25:34,120 --> 04:25:34,680 THOUGHTS, OBSERVATIONS? 6589 04:25:34,680 --> 04:25:44,000 I SEE ONE IN THE BACK. 6590 04:25:44,000 --> 04:25:44,640 6591 04:25:44,640 --> 04:25:45,960 REBECCA IS GETTING A WORKOUT 6592 04:25:45,960 --> 04:25:47,480 RUNNING UP AND DOWN THE STAIRS. 6593 04:25:47,480 --> 04:25:49,560 >> I'M KIM WITH THE CHRISTOPHER 6594 04:25:49,560 --> 04:25:50,880 AND DANA REID FOUNDATION. 6595 04:25:50,880 --> 04:25:54,160 I WANT TO COMMEND YOU FOR YOUR 6596 04:25:54,160 --> 04:25:56,680 EXTRAORDINARY WORK AND I WAS 6597 04:25:56,680 --> 04:26:04,680 CURIOUS ABOUT HOW DO YOU MAN 6598 04:26:04,680 --> 04:26:06,400 EXPECTATIONS, THE APPROVAL 6599 04:26:06,400 --> 04:26:07,520 PROCESS, REIMBURSEMENT PROCESS, 6600 04:26:07,520 --> 04:26:07,800 ACCESS. 6601 04:26:07,800 --> 04:26:12,440 I DON'T WANT TO DEFLATE THESE 6602 04:26:12,440 --> 04:26:13,040 EXTRAORDINARY ACCOMPLISHMENTS 6603 04:26:13,040 --> 04:26:14,960 BUT THIS IS A CONCERN A LOT OF 6604 04:26:14,960 --> 04:26:16,360 ORGANIZATIONS HAVE AND WE'RE 6605 04:26:16,360 --> 04:26:18,800 TALKING ABOUT THAT INTERNALLY AT 6606 04:26:18,800 --> 04:26:20,640 THE FOUNDATION TOO. 6607 04:26:20,640 --> 04:26:23,040 WHEN WE SEE THESE HUGE 6608 04:26:23,040 --> 04:26:24,280 SUCCESSES, AND THE COMMUNITY IS 6609 04:26:24,280 --> 04:26:27,960 DESPERATE TO GET THEM. 6610 04:26:27,960 --> 04:26:28,360 >> GREAT QUESTION. 6611 04:26:28,360 --> 04:26:29,560 THANK YOU SO MUCH. 6612 04:26:29,560 --> 04:26:36,600 I'M GOING TO PASS IT TO MELISSA. 6613 04:26:36,600 --> 04:26:38,720 >> I'M VERY GRATEFUL FOR THE 6614 04:26:38,720 --> 04:26:39,000 QUESTION. 6615 04:26:39,000 --> 04:26:43,760 I DIDN'T SHARE I WAS A 30-YEAR 6616 04:26:43,760 --> 04:26:44,960 HOSPITAL AND HEALTH CARE 6617 04:26:44,960 --> 04:26:46,160 ADMINISTRATOR, LIVED THE PAIN OF 6618 04:26:46,160 --> 04:26:47,320 PAYMENT ISSUES, NOT BEING ABLE 6619 04:26:47,320 --> 04:26:48,800 TO DO THE RIGHT THING FOR 6620 04:26:48,800 --> 04:26:49,560 PATIENTS BECAUSE OF HOW HEALTH 6621 04:26:49,560 --> 04:26:52,280 CARE IS PAID FOR IN OUR COUNTRY. 6622 04:26:52,280 --> 04:26:53,160 SO I APPRECIATE THAT. 6623 04:26:53,160 --> 04:26:54,920 FROM THE VERY BEGINNING OF MY 6624 04:26:54,920 --> 04:26:57,440 TIME WITH THE FOUNDATION I WAS 6625 04:26:57,440 --> 04:26:58,840 INTERESTED IN THE SCIENCE AND 6626 04:26:58,840 --> 04:27:01,440 INTERESTED IN HOW IT WAS 6627 04:27:01,440 --> 04:27:03,040 PROGRESSING, THE COMPOUND WAS 6628 04:27:03,040 --> 04:27:05,160 PROGRESSES, MOST INTERESTED IN 6629 04:27:05,160 --> 04:27:08,760 US PREPARING FOR POST-APPROVAL. 6630 04:27:08,760 --> 04:27:09,960 AND AFFORDABILITY AND ACCESS. 6631 04:27:09,960 --> 04:27:12,680 I WILL SHARE A LITTLE BIT ABOUT 6632 04:27:12,680 --> 04:27:16,840 WHAT WE'RE DOING. 6633 04:27:16,840 --> 04:27:19,600 WE ARE ABOUT 65% OF THE FAMILIES 6634 04:27:19,600 --> 04:27:22,520 WHO HAVE RETT SYNDROME RECEIVE 6635 04:27:22,520 --> 04:27:24,960 THEIR INSURANCE THROUGH PUBLIC 6636 04:27:24,960 --> 04:27:26,840 PAYERS, AND JUST ONE EXAMPLE, 6637 04:27:26,840 --> 04:27:29,480 I'M SURE THERE ARE OTHERS, WE'VE 6638 04:27:29,480 --> 04:27:33,080 MOVED STRONGLY INTO AN ADVOCACY 6639 04:27:33,080 --> 04:27:35,680 WORLD, TEACHING FAMILIES HOW TO 6640 04:27:35,680 --> 04:27:39,600 ADVOCATE FOR COVERAGE NOT 6641 04:27:39,600 --> 04:27:41,880 SPECIFIC TO ONE COMPOUND BUT 6642 04:27:41,880 --> 04:27:43,920 COVERAGE IN GENERAL AT THE STATE 6643 04:27:43,920 --> 04:27:44,120 LEVEL. 6644 04:27:44,120 --> 04:27:47,720 WE'VE ENGAGED WITHED ADVOCACY 6645 04:27:47,720 --> 04:27:49,880 FIRMS TO TEACH FAMILIES TO GO TO 6646 04:27:49,880 --> 04:27:51,800 MEETINGS AT STATE LEVELS, 6647 04:27:51,800 --> 04:27:53,080 PREPARE FOR THOSE CONVERSATIONS, 6648 04:27:53,080 --> 04:27:53,360 ADVOCATE. 6649 04:27:53,360 --> 04:27:55,640 WE HAD A HUGE SUCCESS LAST IF I 6650 04:27:55,640 --> 04:27:58,280 WITH A MOM GOING TO THE MEETING 6651 04:27:58,280 --> 04:28:07,680 IN TEXAS GETTING TEXAS MEDICAID 6652 04:28:07,680 --> 04:28:08,200 TO COVER DAYBUE, YEAH. 6653 04:28:08,200 --> 04:28:10,840 >> IN TERMS OF PREPARING 6654 04:28:10,840 --> 04:28:14,640 EXPECTATIONS, WE WERE VERY 6655 04:28:14,640 --> 04:28:16,520 ADAMANT ABOUT THAT DURING THE 8 6656 04:28:16,520 --> 04:28:17,720 YEARS OF DEVELOPMENT OF THIS 6657 04:28:17,720 --> 04:28:18,040 DRUG. 6658 04:28:18,040 --> 04:28:20,600 WE SAW FROM THE BEGINNING IT 6659 04:28:20,600 --> 04:28:23,320 WOULD NOT BE CURATIVE, WOULD NOT 6660 04:28:23,320 --> 04:28:25,840 BE EVEN PROFOUNDLY BENEFICIAL. 6661 04:28:25,840 --> 04:28:28,880 WE PREPARED OUR PATIENT FAMILIES 6662 04:28:28,880 --> 04:28:31,280 FOR THE IDEA THAT THIS WOULD 6663 04:28:31,280 --> 04:28:32,720 SLOW THE PROGRESSION, IF IT 6664 04:28:32,720 --> 04:28:34,680 WORKED IT WOULD SLOW 6665 04:28:34,680 --> 04:28:37,120 PROGRESSION, GIVE THEIR PATIENTS 6666 04:28:37,120 --> 04:28:38,440 A LITTLE MORE TIME. 6667 04:28:38,440 --> 04:28:40,520 HIGHER QUALITY OF LIFE OVER THAT 6668 04:28:40,520 --> 04:28:45,240 PERIOD OF TIME, WHILE THE MORE 6669 04:28:45,240 --> 04:28:46,120 MAYBE GENE-BASED THERAPIES HAD 6670 04:28:46,120 --> 04:28:48,520 THAT MUCH MORE TIME TO DEVELOP. 6671 04:28:48,520 --> 04:28:51,680 IN TERMS OF THE COVERAGE, WE 6672 04:28:51,680 --> 04:28:54,000 WORKED CLOSELY WITH THE COMPANY 6673 04:28:54,000 --> 04:28:58,040 AND THEY HAVE WORKED AT A 6674 04:28:58,040 --> 04:29:00,440 PROGRAM BOTH PATIENT ASSISTANCE 6675 04:29:00,440 --> 04:29:05,080 TO ASSURE THAT EVERY ELIGIBLE 6676 04:29:05,080 --> 04:29:07,480 PATIENT CAN GAIN ACCESS. 6677 04:29:07,480 --> 04:29:09,760 IF THEY CAN'T AFFORD, YOU KNOW, 6678 04:29:09,760 --> 04:29:11,360 HAVE NO INSURANCE OR ARE 6679 04:29:11,360 --> 04:29:12,040 UNDERINSURED. 6680 04:29:12,040 --> 04:29:13,120 ON THE OTHER HAND, IT'S 6681 04:29:13,120 --> 04:29:15,880 IMPOSSIBLE TO GO TO ALL THE 6682 04:29:15,880 --> 04:29:19,960 PAYERS AND CONVINCE THEM TO 6683 04:29:19,960 --> 04:29:23,080 COVER. 6684 04:29:23,080 --> 04:29:27,360 THAT'S JUST A DAY-TO-DAY TRIAL 6685 04:29:27,360 --> 04:29:28,800 TRYING TO CONVINCE PAYERS AND WE 6686 04:29:28,800 --> 04:29:30,200 DIDN'T HAVE A METHOD OF GOING TO 6687 04:29:30,200 --> 04:29:32,400 ALL PAYERS IN ADVANCE TO PREP 6688 04:29:32,400 --> 04:29:32,600 THEM. 6689 04:29:32,600 --> 04:29:33,840 SO IT'S A TOUGH ONE. 6690 04:29:33,840 --> 04:29:38,440 YOU RAISED A VERY GOOD QUESTION 6691 04:29:38,440 --> 04:29:40,760 >> I AM GOING TO TURN IT OVER TO 6692 04:29:40,760 --> 04:29:40,960 NEIL. 6693 04:29:40,960 --> 04:29:46,720 AND I WANT TO MAKE SURE KIM CAN 6694 04:29:46,720 --> 04:29:46,920 TALK. 6695 04:29:46,920 --> 04:29:48,720 >> I THINK IT'S A REALLY 6696 04:29:48,720 --> 04:29:51,480 IMPORTANT QUESTION. 6697 04:29:51,480 --> 04:29:54,600 AND THE ASSOCIATION WE'VE RAMPED 6698 04:29:54,600 --> 04:29:55,800 UP WITH CMS CONSIDERABLY, THEY 6699 04:29:55,800 --> 04:29:57,440 DON'T HAVE THE PATIENT FOCUS OR 6700 04:29:57,440 --> 04:30:00,280 PROCEDURES IN PLACE TO INCLUDE 6701 04:30:00,280 --> 04:30:02,400 PATIENTS THE WAY THE FDA DOES. 6702 04:30:02,400 --> 04:30:03,440 SO IT'S BEEN MORE OF A 6703 04:30:03,440 --> 04:30:04,240 CHALLENGE. 6704 04:30:04,240 --> 04:30:05,760 AT THE SAME TIME A LOT OF 6705 04:30:05,760 --> 04:30:06,840 INSURANCE DECISIONS ARE MADE ON 6706 04:30:06,840 --> 04:30:08,240 THE STATE LEVEL. 6707 04:30:08,240 --> 04:30:11,760 SO WE'VE BEEN INVOLVED HEAVILY 6708 04:30:11,760 --> 04:30:14,400 ON DIFFERENT INSURANCE ISSUES TO 6709 04:30:14,400 --> 04:30:16,800 COVER MEDI GAP COVERAGE AND 6710 04:30:16,800 --> 04:30:19,640 OTHER VERY COMPLEX FINANCIAL AND 6711 04:30:19,640 --> 04:30:23,360 PAYMENT ISSUES. 6712 04:30:23,360 --> 04:30:24,040 THE UNDERLYING FUNDAMENTALS 6713 04:30:24,040 --> 04:30:25,440 WHETHER A DRUG WORKS OR NOT AND 6714 04:30:25,440 --> 04:30:27,120 HOW TO DELIVER A DRUG TO 6715 04:30:27,120 --> 04:30:28,080 SOMEONE, FIRST YOU HAVE TO 6716 04:30:28,080 --> 04:30:29,160 DIAGNOSE THEM, FIND THEM. 6717 04:30:29,160 --> 04:30:32,440 YOU CAN'T RUN A SUCCESSFUL 6718 04:30:32,440 --> 04:30:33,640 CLINICAL TRIAL ENTERPRISE OR 6719 04:30:33,640 --> 04:30:36,640 TREAT PEOPLE UNLESS YOU GET THEM 6720 04:30:36,640 --> 04:30:37,320 DIAGNOSED. 6721 04:30:37,320 --> 04:30:41,240 ALL OF THOSE QUESTIONS HAVE 6722 04:30:41,240 --> 04:30:42,080 SCIENTIFIC COMPONENTS THAT 6723 04:30:42,080 --> 04:30:43,880 HAVEN'T BEEN FULLY EXPLORED AND 6724 04:30:43,880 --> 04:30:45,280 NEED MORE WORK, AND WE'RE TRYING 6725 04:30:45,280 --> 04:30:48,040 TO BUILD CAPACITY ON THE 6726 04:30:48,040 --> 04:30:49,080 RESEARCH SIDE TO STUDY THESE 6727 04:30:49,080 --> 04:30:51,000 QUESTIONS AS WELL AS DEAL WITH 6728 04:30:51,000 --> 04:30:52,520 THEM AS ADVOCACY ISSUES. 6729 04:30:52,520 --> 04:30:55,560 THEY NEED BOTH LIKE DRUG 6730 04:30:55,560 --> 04:30:56,560 APPROVAL NEEDS ADVOCACY AND 6731 04:30:56,560 --> 04:30:59,080 SCIENTIFIC COMPONENT. 6732 04:30:59,080 --> 04:30:59,960 6733 04:30:59,960 --> 04:31:01,920 >> I CAN JUMP IN BRIEFLY. 6734 04:31:01,920 --> 04:31:08,720 MOST OF IT'S BEEN COVERED. 6735 04:31:08,720 --> 04:31:10,360 WE'LL SEE IT'S JUST BEGINNING TO 6736 04:31:10,360 --> 04:31:11,640 GET OUT, DIAGNOSED WITH A 6737 04:31:11,640 --> 04:31:13,960 GENETIC FORM IS A KEY PART, 6738 04:31:13,960 --> 04:31:15,840 GENETIC TESTING WILL BE MORE 6739 04:31:15,840 --> 04:31:18,480 WIDELY USED IN ALS CLINICS, NOW 6740 04:31:18,480 --> 04:31:21,760 A TREATABLE FORM OF THE DISEASE 6741 04:31:21,760 --> 04:31:23,920 RELATED TO GENETIC FINDINGS. 6742 04:31:23,920 --> 04:31:26,560 AND THEN WE'LL ROLL OUT AND I 6743 04:31:26,560 --> 04:31:28,200 THINK PUSH THIS OUT AND 6744 04:31:28,200 --> 04:31:30,720 HOPEFULLY AS MANY PEOPLE AS 6745 04:31:30,720 --> 04:31:33,160 NEEDED WILL GET ON IT QUICKLY. 6746 04:31:33,160 --> 04:31:34,880 DELAY IS NOT GOOD. 6747 04:31:34,880 --> 04:31:38,680 WE SAW SIX MONTHS OF DELAY IN 6748 04:31:38,680 --> 04:31:42,320 OUR STUDY. 6749 04:31:42,320 --> 04:31:43,960 HIGHLIGHTED SUCCESS OF PEOPLE 6750 04:31:43,960 --> 04:31:45,600 EARLY ON, AND SIX MONTHS DELAYS 6751 04:31:45,600 --> 04:31:47,360 ALSO DID BETTER THAN OTHERWISE, 6752 04:31:47,360 --> 04:31:49,640 THAT'S MY INTERPRETATION OF 6753 04:31:49,640 --> 04:31:49,920 THOSE DATA. 6754 04:31:49,920 --> 04:31:52,680 BUT THEY DIDN'T DO AS WELL. 6755 04:31:52,680 --> 04:31:53,920 TIME REALLY DOES MATTER, 6756 04:31:53,920 --> 04:31:55,080 EVERYONE IN THIS AUDIENCE 6757 04:31:55,080 --> 04:31:56,760 ALREADY KNOWS THIS. 6758 04:31:56,760 --> 04:32:03,960 GETTING PEOPLE ON A DRUG LIKE 6759 04:32:03,960 --> 04:32:06,280 TOFERCIF AND AS IT'S BEEN 6760 04:32:06,280 --> 04:32:07,800 RENAMED AS SOON AS POSSIBLE WILL 6761 04:32:07,800 --> 04:32:10,600 BE KEY TO PRESERVING FUNCTION. 6762 04:32:10,600 --> 04:32:13,080 >> TIME IS NOT ON OUR SIDE. 6763 04:32:13,080 --> 04:32:15,040 I CAN SPEAK IN THE HD SPACE 6764 04:32:15,040 --> 04:32:19,840 WE'RE WAY TOO FAMILIAR WITH 6765 04:32:19,840 --> 04:32:20,080 FAILURE. 6766 04:32:20,080 --> 04:32:21,720 SO MANAGING EXPECTATIONS IS 6767 04:32:21,720 --> 04:32:22,320 HEARTBREAKING. 6768 04:32:22,320 --> 04:32:25,560 WE DO HAVE A QUESTION ONLINE. 6769 04:32:25,560 --> 04:32:25,680 6770 04:32:25,680 --> 04:32:31,160 >> THIS IS A QUESTION FROM JEAN 6771 04:32:31,160 --> 04:32:33,320 SWIDLER FOR DR. MILER. 6772 04:32:33,320 --> 04:32:35,400 DO YOU EXPECT A BETTER RESPONSE 6773 04:32:35,400 --> 04:32:37,920 TO ALS THERAPIES AFTER SYMPTOMS 6774 04:32:37,920 --> 04:32:39,800 OR BEFORE SYMPTOM ONSET IN 6775 04:32:39,800 --> 04:32:42,960 GENETIC ALS? 6776 04:32:42,960 --> 04:32:43,080 6777 04:32:43,080 --> 04:32:44,280 >> HI, JEAN. 6778 04:32:44,280 --> 04:32:46,160 ALWAYS GOOD TO HEAR FROM YOU. 6779 04:32:46,160 --> 04:32:47,440 WE'VE DISCUSSED THIS BEFORE. 6780 04:32:47,440 --> 04:32:50,280 IT'S A VERY GOOD POINT. 6781 04:32:50,280 --> 04:32:53,160 TREATING SOMEBODY AT THE 6782 04:32:53,160 --> 04:32:54,880 EARLIEST POINT POSSIBLE MIGHT 6783 04:32:54,880 --> 04:32:57,680 MEAN TREATING THEM EVEN AT A 6784 04:32:57,680 --> 04:32:58,320 BIOMARKERS CHANGE OR PERHAPS 6785 04:32:58,320 --> 04:33:00,280 BEFORE, THAT AT THE EARLIEST 6786 04:33:00,280 --> 04:33:01,360 INDICATION THAT THE DISEASE IS 6787 04:33:01,360 --> 04:33:01,600 COMING. 6788 04:33:01,600 --> 04:33:04,320 AS SOON AS WE KNOW THAT WITH 6789 04:33:04,320 --> 04:33:08,520 CLARITY, THE DISEASE IS COMING, 6790 04:33:08,520 --> 04:33:10,160 TO GET SOMEBODY ON A DRUG AS 6791 04:33:10,160 --> 04:33:13,240 QUICKLY AS POSSIBLE, TESTED IN 6792 04:33:13,240 --> 04:33:19,160 THE SETTING OF ATLAS TRIAL 6793 04:33:19,160 --> 04:33:20,000 LOOKING FOR BIOMARKERS CHANGE, 6794 04:33:20,000 --> 04:33:21,320 GETTING THEM ON THE DRUG AS SOON 6795 04:33:21,320 --> 04:33:26,160 AS POSSIBLE BEFORE THEY HAVE 6796 04:33:26,160 --> 04:33:27,600 MOTOR MANIFESTATIONS OF ALS, A 6797 04:33:27,600 --> 04:33:29,640 TRIAL FROM BIOGEN AND MICHAEL 6798 04:33:29,640 --> 04:33:29,880 BENATAR. 6799 04:33:29,880 --> 04:33:32,840 AS SOON AS POSSIBLE I THINK IS 6800 04:33:32,840 --> 04:33:33,080 BETTER. 6801 04:33:33,080 --> 04:33:35,480 WE MIGHT BE ABLE TO PREVENT ALS 6802 04:33:35,480 --> 04:33:38,320 ALTOGETHER IF WE CAN GET PEOPLE 6803 04:33:38,320 --> 04:33:41,280 ON A DRUG VERY EARLY. 6804 04:33:41,280 --> 04:33:43,000 AGAIN, THAT'S BEING TESTED. 6805 04:33:43,000 --> 04:33:45,840 AND I WOULD PREDICT THAT THAT'S 6806 04:33:45,840 --> 04:33:47,160 LIKELY TO BE TRUE, THOUGH 6807 04:33:47,160 --> 04:33:50,440 GENERAL IT'S BEING TESTED IN A 6808 04:33:50,440 --> 04:33:52,320 CLINICAL, AND EARLY IS GOING TO 6809 04:33:52,320 --> 04:33:53,760 BE BETTER, I THINK, ALWAYS THAN 6810 04:33:53,760 --> 04:33:56,000 LATER. 6811 04:33:56,000 --> 04:34:00,000 6812 04:34:00,000 --> 04:34:01,600 ONE LAST QUESTION, PENNY? 6813 04:34:01,600 --> 04:34:04,360 >> I'LL MAKE IT SHORT. 6814 04:34:04,360 --> 04:34:06,320 ALL NEUROLOGICAL DISEASE HAS A 6815 04:34:06,320 --> 04:34:08,720 HORRIBLE SUCCESS RATE IN DRUG 6816 04:34:08,720 --> 04:34:11,280 DEVELOPMENT, I WANT TO TAKE A 6817 04:34:11,280 --> 04:34:13,200 MOMENT TO CELEBRATE THERE'S FOUR 6818 04:34:13,200 --> 04:34:15,640 NEW DRUGS FOR RARE NEUROLOGICAL 6819 04:34:15,640 --> 04:34:19,960 DISEASES. 6820 04:34:19,960 --> 04:34:24,160 6821 04:34:24,160 --> 04:34:24,560 6822 04:34:24,560 --> 04:34:28,320 >> LIKE I SAID, I'M JEALOUS. 6823 04:34:28,320 --> 04:34:30,440 WE NEED TO GET BACK ON SCHEDULE, 6824 04:34:30,440 --> 04:34:33,440 I'LL TURN IT OVER TO DR. 6825 04:34:33,440 --> 04:34:33,880 KOROSHETZ. 6826 04:34:33,880 --> 04:34:36,720 I WANT TO THANK THE SPEAKERS. 6827 04:34:36,720 --> 04:34:42,680 NEIL, HOPE YOU FEEL BERT. -- 6828 04:34:42,680 --> 04:34:43,000 BETTER. 6829 04:34:43,000 --> 04:34:44,800 THANK YOU ALL FOR BEING PART OF 6830 04:34:44,800 --> 04:34:45,000 THIS. 6831 04:34:45,000 --> 04:34:48,120 >> THANK YOU VERY MUCH. 6832 04:34:48,120 --> 04:34:55,960 6833 04:34:55,960 --> 04:35:06,040 6834 04:35:32,880 --> 04:35:34,520 >> THAT WAS A FANTASTIC SESSION, 6835 04:35:34,520 --> 04:35:41,160 I MUST SAY. 6836 04:35:41,160 --> 04:35:42,560 ACTUALLY HAVE THREE DRUGS IN A 6837 04:35:42,560 --> 04:35:51,120 YEAR, A RECORD FOR NINDS. 6838 04:35:51,120 --> 04:35:52,440 IT'S A TESTIMONY TO THE HARD 6839 04:35:52,440 --> 04:35:54,400 WORK OF ALL THE PEOPLE WHO MADE 6840 04:35:54,400 --> 04:35:55,840 THIS HAPPEN. 6841 04:35:55,840 --> 04:36:03,280 HERE AT NINDS WE LOOK AT THE BIG 6842 04:36:03,280 --> 04:36:04,160 PICTURE. 6843 04:36:04,160 --> 04:36:05,800 AND I'M HOPING THAT AS NEIL SAID 6844 04:36:05,800 --> 04:36:08,520 THERE IS GOING TO BE MORE AND 6845 04:36:08,520 --> 04:36:14,640 MORE THERAPIES COMING OUT, MORE 6846 04:36:14,640 --> 04:36:15,520 SUCCESSFUL, EVERY YEAR. 6847 04:36:15,520 --> 04:36:18,640 JUST TO SET THE STAGE, HASN'T 6848 04:36:18,640 --> 04:36:26,160 ALWAYS BEEN THIS GOOD. 6849 04:36:26,160 --> 04:36:31,520 SO WE, AS PENNY SAID, A LOT OF 6850 04:36:31,520 --> 04:36:33,720 TRIALS DIDN'T WORK AT ALL. 6851 04:36:33,720 --> 04:36:37,200 USUALLY THE REASON IS BECAUSE WE 6852 04:36:37,200 --> 04:36:38,400 MADE THESE ASSUMPTION ABOUT THE 6853 04:36:38,400 --> 04:36:41,440 DRUG OR ABOUT THE DISEASE, AND 6854 04:36:41,440 --> 04:36:43,560 WE WERE WRONG, THOSE 6855 04:36:43,560 --> 04:36:45,840 ASSUMPTIONS, AND THEY CAME BACK 6856 04:36:45,840 --> 04:36:47,400 TO BITE US. 6857 04:36:47,400 --> 04:36:50,040 AT NINDS WHAT WE TRY TO DO IS 6858 04:36:50,040 --> 04:36:54,840 KIND OF LIKE YOU INVEST IN YOUR 6859 04:36:54,840 --> 04:36:55,840 PORTFOLIO, YOUR FINANCIAL 6860 04:36:55,840 --> 04:36:59,320 PORTFOLIO, IN TERMS OF ACROSS 6861 04:36:59,320 --> 04:37:01,120 THE SPECTRUM OF ACTIVITIES, AND 6862 04:37:01,120 --> 04:37:02,400 ALWAYS TRYING TO KEEP OUR EYE ON 6863 04:37:02,400 --> 04:37:11,720 THE BALL IN TERMS OF THE HEALTH 6864 04:37:11,720 --> 04:37:13,440 OF THE DIFFERENT PARTS OF THE 6865 04:37:13,440 --> 04:37:13,680 PUZZLE. 6866 04:37:13,680 --> 04:37:16,960 WE ALSO WATCH WHAT YOU DO, WE 6867 04:37:16,960 --> 04:37:18,840 TRY AND BE COMPLEMENTARY, LOOK 6868 04:37:18,840 --> 04:37:23,960 AT WHAT THE INDUSTRY DOES, TRY 6869 04:37:23,960 --> 04:37:25,480 AND MAKE THEIR CHANCE OF SUCCESS 6870 04:37:25,480 --> 04:37:27,040 GREATER AS THEY GO INTO THESE 6871 04:37:27,040 --> 04:37:35,760 THINGS AND TRY TO PULL INDUSTRY 6872 04:37:35,760 --> 04:37:35,920 IN. 6873 04:37:35,920 --> 04:37:37,240 I'M GOING TO TALK ABOUT THE 6874 04:37:37,240 --> 04:37:38,560 INSTITUTE IN GENERAL. 6875 04:37:38,560 --> 04:37:41,360 SO, TO THE POINT I JUST MADE, IN 6876 04:37:41,360 --> 04:37:44,800 TERMS OF INVESTING ACROSS THE 6877 04:37:44,800 --> 04:37:47,400 SPECTRUM, WE HAVE THESE THREE 6878 04:37:47,400 --> 04:37:52,160 DIVISIONS OF SCIENCE, DIVISION 6879 04:37:52,160 --> 04:37:57,880 OF NEUROSCIENCE, DIVISION OF 6880 04:37:57,880 --> 04:38:00,080 TRANSLATIONAL RESEARCH, I DON'T 6881 04:38:00,080 --> 04:38:06,200 SEE AMIR HERE, AND PEOPLE FROM 6882 04:38:06,200 --> 04:38:12,560 DTR, AND DIVISION OF CLINICAL 6883 04:38:12,560 --> 04:38:13,120 RESEARCH. 6884 04:38:13,120 --> 04:38:14,840 AND WE INVEST HEAVILY ON THE 6885 04:38:14,840 --> 04:38:16,040 BASIC SIDE TO UNDERSTAND 6886 04:38:16,040 --> 04:38:20,120 MECHANISM OF DISEASE. 6887 04:38:20,120 --> 04:38:23,400 IN FACT, PROBABLY ABOUT 65% OF 6888 04:38:23,400 --> 04:38:27,560 OUR MONEY GOES TO THIS BASIC 6889 04:38:27,560 --> 04:38:28,080 RESEARCH. 6890 04:38:28,080 --> 04:38:30,280 ABOUT 22% OF OUR MONEY GOES TO 6891 04:38:30,280 --> 04:38:33,040 UNDERSTANDING HOW THE BRAIN 6892 04:38:33,040 --> 04:38:36,760 WORKS IN GENERAL. 6893 04:38:36,760 --> 04:38:38,280 THAT'S DISEASE AGNOSTIC. 6894 04:38:38,280 --> 04:38:41,120 ABOUT 50% GOES TO BASIC 6895 04:38:41,120 --> 04:38:42,680 MECHANISMS OF DIFFERENT 6896 04:38:42,680 --> 04:38:43,000 DISEASES. 6897 04:38:43,000 --> 04:38:46,480 AND WE HAVE HUNDREDS OF DISEASES 6898 04:38:46,480 --> 04:38:49,120 IN OUR PORTFOLIO, SO THERE'S NO 6899 04:38:49,120 --> 04:38:51,320 DISEASE THAT GETS ENOUGH MONEY. 6900 04:38:51,320 --> 04:38:53,120 IT'S SPREAD OUT DEPENDING HOW 6901 04:38:53,120 --> 04:38:57,360 YOU COUNT NOW, MEASURE UP TO 400 6902 04:38:57,360 --> 04:38:59,160 DIFFERENT NEUROLOGIC DISEASES. 6903 04:38:59,160 --> 04:39:01,080 AND DIVISION OF CLINICAL 6904 04:39:01,080 --> 04:39:02,400 RESEARCH DOES CLINICAL TRIALS. 6905 04:39:02,400 --> 04:39:03,840 BUT IN BETWEEN WE HAVE THIS 6906 04:39:03,840 --> 04:39:05,760 GROUP CALLED DIVISION OF 6907 04:39:05,760 --> 04:39:07,320 TRANSLATIONAL RESEARCH, THE 6908 04:39:07,320 --> 04:39:11,800 PURPOSE OF THAT GROUP IS TO 6909 04:39:11,800 --> 04:39:14,760 FACILITATE DEVELOPMENT OF NEW 6910 04:39:14,760 --> 04:39:15,040 THERAPIES. 6911 04:39:15,040 --> 04:39:20,360 AND WE ARE NOT A DRUG COMPANY. 6912 04:39:20,360 --> 04:39:22,560 BUT WE RELY ON DRUG COMPANIES TO 6913 04:39:22,560 --> 04:39:24,080 TAKE ADVANCING OUT OF THE 6914 04:39:24,080 --> 04:39:26,960 RESEARCH WE FUND IN THESE BASIC 6915 04:39:26,960 --> 04:39:30,560 MECHANISMS OF DISEASE TO BRING 6916 04:39:30,560 --> 04:39:31,840 PRODUCTS TO PEOPLE. 6917 04:39:31,840 --> 04:39:34,280 SO NOW IN OUR BUSINESS THERE ARE 6918 04:39:34,280 --> 04:39:36,560 SOME DISEASES THAT HAVE A LOT OF 6919 04:39:36,560 --> 04:39:37,480 INDUSTRY INTEREST. 6920 04:39:37,480 --> 04:39:39,200 IT TENDS TO WAX AND WANE. 6921 04:39:39,200 --> 04:39:41,480 WE HAVE A LOT OF DISEASES THAT 6922 04:39:41,480 --> 04:39:43,560 HAVE NO INDUSTRY INTEREST. 6923 04:39:43,560 --> 04:39:46,680 AND THERE WHAT WE TRY TO DO IS 6924 04:39:46,680 --> 04:39:49,080 DEVELOP THINGS SO THAT IT LOOKS 6925 04:39:49,080 --> 04:39:51,280 LESS RISKY. 6926 04:39:51,280 --> 04:39:52,360 USUALLY THE COMPANIES DON'T -- 6927 04:39:52,360 --> 04:39:53,800 IT'S NOT THAT THEY DON'T FEEL 6928 04:39:53,800 --> 04:39:55,760 THEY CAN MAKE MONEY. 6929 04:39:55,760 --> 04:39:58,040 THE PROBLEM IS THAT THE RISK OF 6930 04:39:58,040 --> 04:40:05,400 FAILURE IS SO HIGH, THAT'S 6931 04:40:05,400 --> 04:40:06,680 BECAUSE OF INHERENT PROBLEM 6932 04:40:06,680 --> 04:40:07,880 DEVELOPING DRUGS FOR NERVOUS 6933 04:40:07,880 --> 04:40:08,560 SYSTEM DISEASES. 6934 04:40:08,560 --> 04:40:12,600 THERE'S A HIGH FAILURE RATE, 6935 04:40:12,600 --> 04:40:14,480 ASPENY MENTIONED, WE THINK 6936 04:40:14,480 --> 04:40:25,000 THAT'S RELATED TO OUR LACK OF 6937 04:40:28,880 --> 04:40:29,080 KNOWLEDGE. 6938 04:40:29,080 --> 04:40:30,240 CANCER TRIES TO KILL CELLS, 6939 04:40:30,240 --> 04:40:34,200 WE'RE TRYING TO KEEP CELLS 6940 04:40:34,200 --> 04:40:34,720 ALIVE. 6941 04:40:34,720 --> 04:40:36,520 IT'S NOT IMPOSSIBLE. 6942 04:40:36,520 --> 04:40:40,000 TAKES A LOT OF HARD WORK, A 6943 04:40:40,000 --> 04:40:42,480 LITTLE LUCK. 6944 04:40:42,480 --> 04:40:44,040 FROM RON'S SAGA DECADES TO GET 6945 04:40:44,040 --> 04:40:46,040 THERE BUT IT'S ALL WORTH IT 6946 04:40:46,040 --> 04:40:48,960 BECAUSE OF THE IMPORTANT IMPACT 6947 04:40:48,960 --> 04:40:50,400 THESE THERAPIES HAVE. 6948 04:40:50,400 --> 04:40:51,160 DIVISION OF TRANSLATIONAL 6949 04:40:51,160 --> 04:40:54,680 RESEARCH IS PEOPLE WHO CAME IN 6950 04:40:54,680 --> 04:40:55,760 FROM PHARMACEUTICAL COMPANIES, 6951 04:40:55,760 --> 04:40:57,960 WHEN MOST COMPANIES DROPPED 6952 04:40:57,960 --> 04:40:58,560 NEUROSCIENCE PROGRAMS BECAUSE 6953 04:40:58,560 --> 04:41:06,160 THEY FELT THEY WERE TOO RISKY. 6954 04:41:06,160 --> 04:41:08,360 SO THAT'S KIND OF OUR GENERAL 6955 04:41:08,360 --> 04:41:09,240 OVERALL LOOK AT THINGS. 6956 04:41:09,240 --> 04:41:13,200 IF YOU LOOK AT WHAT INDUSTRY 6957 04:41:13,200 --> 04:41:14,480 DOES, IT'S A PYRAMID, EXACTLY 6958 04:41:14,480 --> 04:41:15,600 THE OPPOSITE OF OURS. 6959 04:41:15,600 --> 04:41:17,880 MOST OF THEIR MONEY WILL GO INTO 6960 04:41:17,880 --> 04:41:19,720 THESE BIG EXPENSIVE CLINICAL 6961 04:41:19,720 --> 04:41:20,520 TRIALS. 6962 04:41:20,520 --> 04:41:23,120 GETTING THINGS TO FDA APPROVAL. 6963 04:41:23,120 --> 04:41:26,640 MOST OF OUR MONEY IS 6964 04:41:26,640 --> 04:41:28,720 UNDERPINNING THAT, AND WITHOUT 6965 04:41:28,720 --> 04:41:30,560 US THERE WOULDN'T BE THE DISEASE 6966 04:41:30,560 --> 04:41:32,120 RESEARCH THAT'S NEEDED FOR THE 6967 04:41:32,120 --> 04:41:33,760 INDUSTRY TO MOVE FORWARD. 6968 04:41:33,760 --> 04:41:34,960 IT'S VERY COMPLEMENTARY TO WHAT 6969 04:41:34,960 --> 04:41:36,400 THE INDUSTRY DOES. 6970 04:41:36,400 --> 04:41:39,360 WE WORK WITH THEM CLOSELY. 6971 04:41:39,360 --> 04:41:41,320 WE HAVE A NUMBER OF 6972 04:41:41,320 --> 04:41:42,560 PUBLIC/PRIVATE PARTNERSHIPS, 6973 04:41:42,560 --> 04:41:44,960 ALWAYS INTERESTED IN DISCUSSING 6974 04:41:44,960 --> 04:41:46,800 THINGS WITH INDUSTRY. 6975 04:41:46,800 --> 04:41:48,760 WE HAVE A COUPLE PROGRAMS REALLY 6976 04:41:48,760 --> 04:41:50,720 FOCUSED ON INDUSTRY TO MOVE 6977 04:41:50,720 --> 04:41:56,120 THINGS FORWARD, MOSTLY SMALL 6978 04:41:56,120 --> 04:41:58,960 BIOTECHS, SMALL BUSINESS GRANTS. 6979 04:41:58,960 --> 04:42:02,560 OOPS, WRONG WAY. 6980 04:42:02,560 --> 04:42:04,640 AND WE ALSO HAVE THE INTRAMURAL 6981 04:42:04,640 --> 04:42:08,360 PROGRAM, YOU HEARD FROM REGHAN 6982 04:42:08,360 --> 04:42:10,360 FOLEY TODAY, THAT'S ANOTHER 6983 04:42:10,360 --> 04:42:12,600 SPECIAL PLACE BECAUSE THERE'S A 6984 04:42:12,600 --> 04:42:13,360 RESEARCH HOSPITAL. 6985 04:42:13,360 --> 04:42:17,880 IF YOU TRY AND DO RESEARCH OUT 6986 04:42:17,880 --> 04:42:21,720 IN THE EXTRAMURAL SPACE, 6987 04:42:21,720 --> 04:42:22,560 INCREDIBLY EXPENSIVE, AND 6988 04:42:22,560 --> 04:42:24,280 CERTAINLY VERY HARD TO DO 6989 04:42:24,280 --> 04:42:25,880 ANYTHING THAT REQUIRES SOMEBODY 6990 04:42:25,880 --> 04:42:28,280 ACTUALLY STAYING IN THE HOSPITAL 6991 04:42:28,280 --> 04:42:28,680 OVERNIGHT. 6992 04:42:28,680 --> 04:42:32,240 MOST OF THE TRIALS YOU HEARD 6993 04:42:32,240 --> 04:42:33,600 ABOUT ARE BASICALLY OUTPATIENT 6994 04:42:33,600 --> 04:42:34,440 TRIALS, BUT THERE'S SOME THINGS 6995 04:42:34,440 --> 04:42:36,080 WHERE YOU NEED TO BRING PEOPLE 6996 04:42:36,080 --> 04:42:39,480 IN, KEEP THEM IN FOR WEEKS, 6997 04:42:39,480 --> 04:42:40,560 SOMETIMES AT A TIME. 6998 04:42:40,560 --> 04:42:42,120 CERTAINLY THE GENE THERAPY 6999 04:42:42,120 --> 04:42:43,520 SPACE, THAT'S LIKELY TO BE THE 7000 04:42:43,520 --> 04:42:45,280 CASE, CERTAINLY IN THE 7001 04:42:45,280 --> 04:42:45,560 BEGINNING. 7002 04:42:45,560 --> 04:42:48,240 SO THE HOSPITAL HERE CAN 7003 04:42:48,240 --> 04:42:49,640 ACTUALLY DO THAT. 7004 04:42:49,640 --> 04:42:52,720 IT'S NOT FUNDED ON GRANTS. 7005 04:42:52,720 --> 04:42:55,560 THE PEOPLE IN THE INTRAMURAL 7006 04:42:55,560 --> 04:42:56,640 PROGRAM ARE FEDERAL EMPLOYEES. 7007 04:42:56,640 --> 04:42:59,400 AND THEY HAVE A SALARY AND THEY 7008 04:42:59,400 --> 04:43:01,480 HAVE A BUDGET THAT THEY CAN 7009 04:43:01,480 --> 04:43:03,440 COUNT ON MOSTLY FROM YEAR TO 7010 04:43:03,440 --> 04:43:05,160 YEAR, DEPENDING WHAT CONGRESS 7011 04:43:05,160 --> 04:43:05,920 DOES. 7012 04:43:05,920 --> 04:43:07,560 IT'S A VERY DIFFERENT ATMOSPHERE 7013 04:43:07,560 --> 04:43:09,760 THAN ON THE OUTSIDE WHERE 7014 04:43:09,760 --> 04:43:13,760 THERE'S A WHOLE BUNCH OF 7015 04:43:13,760 --> 04:43:14,680 COMPETING INTERESTS. 7016 04:43:14,680 --> 04:43:17,840 SO OUR PROGRAM, ON THE 7017 04:43:17,840 --> 04:43:19,200 INTRAMURAL SIDE, HAS HIGH 7018 04:43:19,200 --> 04:43:20,200 QUALITY BUT IT'S FOCUSED IN 7019 04:43:20,200 --> 04:43:22,040 PARTICULAR AREAS, NOT LIKE A 7020 04:43:22,040 --> 04:43:25,640 GENERAL HOSPITAL WHERE THEY HAVE 7021 04:43:25,640 --> 04:43:26,840 PROGRAMS IN ALL DISEASES, THEY 7022 04:43:26,840 --> 04:43:30,240 HAVE I WOULD SAY COMPARED TO 7023 04:43:30,240 --> 04:43:32,480 WHERE I CAME FROM MIGHT COVER 7024 04:43:32,480 --> 04:43:34,200 15% OF DISEASES THAT A GENERAL 7025 04:43:34,200 --> 04:43:36,600 HOSPITAL WILL COVER BUT THEY CAN 7026 04:43:36,600 --> 04:43:38,920 DO THINGS YOU CAN'T DO IN A 7027 04:43:38,920 --> 04:43:41,440 GENERAL HOSPITAL. 7028 04:43:41,440 --> 04:43:47,440 7029 04:43:47,440 --> 04:43:48,960 7030 04:43:48,960 --> 04:43:51,400 HOW DO WE MAKE FUNDING 7031 04:43:51,400 --> 04:43:52,600 DECISIONS? 7032 04:43:52,600 --> 04:43:53,640 NEIL MENTIONED CANCER INSTITUTE, 7033 04:43:53,640 --> 04:43:57,400 WHICH DOES A LOT OF TOP-DOWN 7034 04:43:57,400 --> 04:43:57,640 FUNDING. 7035 04:43:57,640 --> 04:44:03,320 AND WE TEND TO BE THE OPPOSITE, 7036 04:44:03,320 --> 04:44:05,400 A LOT OF BOTTOM-UP. 7037 04:44:05,400 --> 04:44:06,680 I'M NOT SAYING WE'RE BETTER. 7038 04:44:06,680 --> 04:44:08,120 WE HAVE TO THINK ABOUT WHERE WE 7039 04:44:08,120 --> 04:44:10,920 ARE AND HOW WE DO THIS. 7040 04:44:10,920 --> 04:44:16,160 YOU CAN SEE THIS DARK BLUE AREA 7041 04:44:16,160 --> 04:44:16,920 CALLED INVESTIGATOR INITIATED 7042 04:44:16,920 --> 04:44:20,200 RESEARCH, SO WHAT THIS MEANS IS 7043 04:44:20,200 --> 04:44:21,080 THESE ARE RESEARCH OPPORTUNITIES 7044 04:44:21,080 --> 04:44:24,480 THAT ARE OPEN FOR ANYBODY TO 7045 04:44:24,480 --> 04:44:27,760 COME IN, AND SUBMIT GRANTS, GET 7046 04:44:27,760 --> 04:44:29,200 REVIEWED, GET A SCORE, PAY THE 7047 04:44:29,200 --> 04:44:32,240 GOOD SCORES UNTIL WE RUN OUT OF 7048 04:44:32,240 --> 04:44:34,840 MONEY, AND THAT'S HOW, AS YOU 7049 04:44:34,840 --> 04:44:38,480 CAN SEE HERE, THAT'S 60, 70% OF 7050 04:44:38,480 --> 04:44:40,320 OUR MONEY. 7051 04:44:40,320 --> 04:44:42,680 IT'S REALLY NOT TOP-DOWN. 7052 04:44:42,680 --> 04:44:45,280 WHEN YOU HAVE 400 DISEASES, 7053 04:44:45,280 --> 04:44:51,160 PLAYING FAVORITES IS VERY TRICKY 7054 04:44:51,160 --> 04:44:52,800 FOR ME ANYWAY. 7055 04:44:52,800 --> 04:44:54,640 I HATE MY DISEASES AND LOVE THEM 7056 04:44:54,640 --> 04:44:56,080 ALL EQUALLY. 7057 04:44:56,080 --> 04:44:59,800 WHAT THIS DOES, BASICALLY IS 7058 04:44:59,800 --> 04:45:00,240 COMPETITION. 7059 04:45:00,240 --> 04:45:01,880 SO, THAT ACCOUNTS FOR A LOT OF 7060 04:45:01,880 --> 04:45:02,200 DISPARITIES. 7061 04:45:02,200 --> 04:45:03,280 WE HAVE SOME RESEARCH WHERE A 7062 04:45:03,280 --> 04:45:04,520 LOT OF PEOPLE COME IN. 7063 04:45:04,520 --> 04:45:06,160 THERE'S A LOT OF PROGRESS THEY 7064 04:45:06,160 --> 04:45:07,480 THINK THEY CAN MAKE, THEY ARE 7065 04:45:07,480 --> 04:45:09,640 GOING TO GET GRANTS, THAT MONEY 7066 04:45:09,640 --> 04:45:12,840 RISES IN THAT AREA OF RESEARCH. 7067 04:45:12,840 --> 04:45:15,480 THERE ARE ALSO AREAS STRUGGLING 7068 04:45:15,480 --> 04:45:17,000 TO PULL PEOPLE IN BECAUSE THEY 7069 04:45:17,000 --> 04:45:18,320 DON'T HAVE THE TOOLS, PEOPLE 7070 04:45:18,320 --> 04:45:21,120 CAN'T FIGURE OUT EXACTLY HOW TO 7071 04:45:21,120 --> 04:45:23,360 GET AT THIS CONDITION, AND SO, 7072 04:45:23,360 --> 04:45:25,080 YEAH, THOSE CONDITIONS SUFFER. 7073 04:45:25,080 --> 04:45:27,400 AND WE LOOK FOR THOSE AND 7074 04:45:27,400 --> 04:45:30,000 SOMETIMES WILL PUT OUT A 7075 04:45:30,000 --> 04:45:31,560 PARTICULAR CALL FOR GRANTS IN 7076 04:45:31,560 --> 04:45:32,200 THIS SPACE. 7077 04:45:32,200 --> 04:45:34,720 I MUST SAY THAT DOESN'T WORK 7078 04:45:34,720 --> 04:45:35,600 VERY WELL. 7079 04:45:35,600 --> 04:45:37,360 PEOPLE THINK THAT BY PUTTING 7080 04:45:37,360 --> 04:45:38,440 MONEY ON THE STREET THAT'S GOING 7081 04:45:38,440 --> 04:45:41,720 TO MAKE IT HAPPEN. 7082 04:45:41,720 --> 04:45:42,920 THAT'S NOT TRUE. 7083 04:45:42,920 --> 04:45:44,800 PEOPLE WILL RESEARCH WHAT THEY 7084 04:45:44,800 --> 04:45:45,360 ARE PASSIONATE ABOUT 7085 04:45:45,360 --> 04:45:45,880 RESEARCHING. 7086 04:45:45,880 --> 04:45:47,880 IF THEY ARE NOT PASSIONATE, THEY 7087 04:45:47,880 --> 04:45:50,200 WILL SEE WHAT THEY ARE 7088 04:45:50,200 --> 04:45:51,320 PASSIONATE ABOUT. 7089 04:45:51,320 --> 04:45:53,280 WHAT WE RELY ON IS PEOPLE IN THE 7090 04:45:53,280 --> 04:45:58,960 ROOM AND ON ZOOM TO REALLY HOOK 7091 04:45:58,960 --> 04:46:01,240 PEOPLE ON NEURODISEASE, MAKE 7092 04:46:01,240 --> 04:46:03,680 THEM PASSIONATE RESEARCHERS, 7093 04:46:03,680 --> 04:46:05,000 CLINICAL TRIALISTS, BASIC 7094 04:46:05,000 --> 04:46:10,000 SCIENTISTS TO PUSH HARD IN THAT 7095 04:46:10,000 --> 04:46:10,440 DISEASE. 7096 04:46:10,440 --> 04:46:11,560 GOVERNMENT PUTTING OUT MONEY 7097 04:46:11,560 --> 04:46:14,280 DOESN'T REALLY DO THE TRICK IN 7098 04:46:14,280 --> 04:46:17,680 TERMS OF GETTING REALLY GOOD 7099 04:46:17,680 --> 04:46:19,320 PEOPLE TO COME INTO AN AREA. 7100 04:46:19,320 --> 04:46:22,040 NOW, WE DO CERTAIN THINGS WHERE 7101 04:46:22,040 --> 04:46:25,560 WE TRY AND PULL A LIKE AREA IN 7102 04:46:25,560 --> 04:46:29,720 TO ANOTHER AREA THAT'S NOT DOING 7103 04:46:29,720 --> 04:46:32,880 AS WELL. 7104 04:46:32,880 --> 04:46:34,360 BUT IT'S MORE KIND OF, YOU KNOW, 7105 04:46:34,360 --> 04:46:36,760 ICING AROUND THE CAKE AS OPPOSED 7106 04:46:36,760 --> 04:46:39,160 TO THE REAL MEAT OF THE PROBLEM. 7107 04:46:39,160 --> 04:46:42,280 SO WHAT YOU DO IN ATTRACTING 7108 04:46:42,280 --> 04:46:44,000 PEEL 7109 04:46:44,000 --> 04:46:46,240 PEOPLE TO WORK IN THE DISEASES, 7110 04:46:46,240 --> 04:46:47,960 YOU CAN'T IMAGINE HOW IMPORTANT 7111 04:46:47,960 --> 04:46:48,560 IT IS. 7112 04:46:48,560 --> 04:46:49,720 IT IS DISEASE ORGANIZATIONS THAT 7113 04:46:49,720 --> 04:46:51,600 HAVE THE IMPACT ON THE YOUNG 7114 04:46:51,600 --> 04:46:52,680 SCIENTISTS, IN TERMS OF WHAT 7115 04:46:52,680 --> 04:46:56,720 THEY ARE GOING TO STUDY. 7116 04:46:56,720 --> 04:46:58,040 WE ALSO PUT MONEY, OTHER THINGS 7117 04:46:58,040 --> 04:47:00,800 YOU CAN SEE, PUT MONEY INTO 7118 04:47:00,800 --> 04:47:01,120 TRAINING. 7119 04:47:01,120 --> 04:47:03,120 WE HAVE NETWORKS FOR CLINICAL 7120 04:47:03,120 --> 04:47:03,960 TRIALS. 7121 04:47:03,960 --> 04:47:05,080 WE HAVE -- THESE ARE NETWORKS 7122 04:47:05,080 --> 04:47:08,880 FOR TRIALS THAT ARE OCCURRING AT 7123 04:47:08,880 --> 04:47:09,960 FAIRLY HIGH VOLUME. 7124 04:47:09,960 --> 04:47:11,400 YOU WOULDN'T SET A NETWORK UP 7125 04:47:11,400 --> 04:47:13,680 FOR A TRIAL WHERE YOU DO ONE 7126 04:47:13,680 --> 04:47:14,920 EVERY FIVE YEARS. 7127 04:47:14,920 --> 04:47:16,640 BUT IF YOU'RE DOING FIVE TRIALS 7128 04:47:16,640 --> 04:47:17,520 SIMULTANEOUSLY THAT WOULD BE A 7129 04:47:17,520 --> 04:47:20,840 GOOD PLACE TO DO A NETWORK. 7130 04:47:20,840 --> 04:47:23,000 THE FIRST NETWORK WE PUT 7131 04:47:23,000 --> 04:47:24,760 TOGETHER WAS FOR EMERGENCY 7132 04:47:24,760 --> 04:47:25,040 RESEARCH. 7133 04:47:25,040 --> 04:47:29,240 AND THAT WENT REALLY WELL. 7134 04:47:29,240 --> 04:47:30,960 SECOND WAS CALLED NEURONEXT, 7135 04:47:30,960 --> 04:47:33,400 PHASE 2 FIRST-IN-HUMAN TRIALS, 7136 04:47:33,400 --> 04:47:34,720 ANY NEUROLOGICAL CONDITION. 7137 04:47:34,720 --> 04:47:35,720 THAT ALSO WORKED WELL, 7138 04:47:35,720 --> 04:47:38,320 STRUGGLING A LITTLE NOW TO GET 7139 04:47:38,320 --> 04:47:43,040 GOOD APPLICATIONS IN. 7140 04:47:43,040 --> 04:47:45,080 WE THINK BECAUSE OF TREATMENTS 7141 04:47:45,080 --> 04:47:46,640 COMING OUT NEURONEXT WAS GOING 7142 04:47:46,640 --> 04:47:49,400 TO SERVE A GREAT PURPOSE. 7143 04:47:49,400 --> 04:47:52,880 WE HAVE A NETWORK FOR STROKE 7144 04:47:52,880 --> 04:47:54,960 TRIALS, S IN NINDS STANDS FOR 7145 04:47:54,960 --> 04:47:55,520 STROKE. 7146 04:47:55,520 --> 04:47:57,160 IT IS THE THIRD -- FOURTH 7147 04:47:57,160 --> 04:47:59,800 LEADING CAUSE OF DEATH IN THE 7148 04:47:59,800 --> 04:48:00,120 U.S. 7149 04:48:00,120 --> 04:48:03,520 AND THERE IS NO COMPANY IN THE 7150 04:48:03,520 --> 04:48:05,840 COUNTRY INTERESTED IN STROKE. 7151 04:48:05,840 --> 04:48:11,160 THAT'S A REALLY COMMON DISEASE, 7152 04:48:11,160 --> 04:48:11,840 KILLS PEOPLE, CAUSES GREAT 7153 04:48:11,840 --> 04:48:13,000 DISABILITY, AND NO COMPANY 7154 04:48:13,000 --> 04:48:13,360 INTEREST. 7155 04:48:13,360 --> 04:48:15,880 THAT'S NOT BECAUSE THEY CAN'T 7156 04:48:15,880 --> 04:48:17,160 MAKE MONEY BUT BECAUSE THEY HAVE 7157 04:48:17,160 --> 04:48:19,000 SPENT SO MANY BILLIONS OF 7158 04:48:19,000 --> 04:48:25,640 DOLLARS IN SALES IN THE 199 7159 04:48:25,640 --> 04:48:26,600 0s, EARLY 2000s. 7160 04:48:26,600 --> 04:48:29,760 HAVING BEEN A STROKE PERSON THAT 7161 04:48:29,760 --> 04:48:31,160 FAILED, IF YOU GO INTO SOMETHING 7162 04:48:31,160 --> 04:48:33,720 AND YOU RUN A BIG PROGRAM AND 7163 04:48:33,720 --> 04:48:36,240 YOU RUN TEN TRIALS AND THEY ALL 7164 04:48:36,240 --> 04:48:37,480 FAIL, THE COMPANIES WILL LEAVE 7165 04:48:37,480 --> 04:48:41,120 AND THEY ARE NOT COMING BACK FOR 7166 04:48:41,120 --> 04:48:43,600 ANOTHER 10, 20 YEARS, IF YOU'RE 7167 04:48:43,600 --> 04:48:43,800 LUCKY. 7168 04:48:43,800 --> 04:48:45,800 SO FAILED TRIAL IS A BAD THING. 7169 04:48:45,800 --> 04:48:48,080 TEN FAILED TRIALS COULD BE A 7170 04:48:48,080 --> 04:48:49,320 TOTAL DISASTER TO A FIELD. 7171 04:48:49,320 --> 04:48:51,400 YOU'VE GOT TO BE VERY CAREFUL US 7172 04:48:51,400 --> 04:48:53,400 A MOVE INTO THESE SPACES. 7173 04:48:53,400 --> 04:48:58,840 THE COMPANIES, YOU WATCH THEM, 7174 04:48:58,840 --> 04:49:00,120 VERY MUCH LIKE LEMMINGS, MOVE IN 7175 04:49:00,120 --> 04:49:01,640 A GROUP, WILL JUMP OFF A CLIFF 7176 04:49:01,640 --> 04:49:02,640 IN A GROUP. 7177 04:49:02,640 --> 04:49:04,400 BUT THEY ARE SMARTER, THEY WON'T 7178 04:49:04,400 --> 04:49:07,480 JUMP OFF A CLIFF TWICE. 7179 04:49:07,480 --> 04:49:11,840 YOU WANT TO WORK CAREFULLY IN 7180 04:49:11,840 --> 04:49:14,040 THIS SPACE, JUST BECAUSE OF 7181 04:49:14,040 --> 04:49:16,120 THOSE EXPERIENCES. 7182 04:49:16,120 --> 04:49:17,960 THAT'S HOW YOU GET A COMPANY -- 7183 04:49:17,960 --> 04:49:20,040 A COMPANY DROPS IN THE AREA OF 7184 04:49:20,040 --> 04:49:22,760 SCIENCE, BECAUSE THEY HAVE LOST 7185 04:49:22,760 --> 04:49:24,640 SO MUCH MONEY TRYING TO TEST 7186 04:49:24,640 --> 04:49:26,440 THERAPY IN A PHASE 3 TRIAL AND 7187 04:49:26,440 --> 04:49:34,760 THEN THE PHASE 3 TRIAL FAILS. 7188 04:49:34,760 --> 04:49:37,400 WE HAVE BEEN REALLY FORTUNATE TO 7189 04:49:37,400 --> 04:49:38,880 HAVE RECEIVED A LOT OF TAXPAYER 7190 04:49:38,880 --> 04:49:42,880 MONEY OVER THE LAST COUPLE 7191 04:49:42,880 --> 04:49:43,080 YEARS. 7192 04:49:43,080 --> 04:49:45,160 BASICALLY SINCE I BECAME 7193 04:49:45,160 --> 04:49:47,320 DIRECTOR OUR BUDGET HAS PROBABLY 7194 04:49:47,320 --> 04:49:47,560 DOUBLED. 7195 04:49:47,560 --> 04:49:52,560 WE MANAGED NOW CLOSE TO $3 7196 04:49:52,560 --> 04:49:54,840 BILLION, AND THE THING ABOUT THE 7197 04:49:54,840 --> 04:49:57,480 MONEY IS THAT ABOUT A BILLION OF 7198 04:49:57,480 --> 04:49:59,200 THOSE DOLLARS ARE 7199 04:49:59,200 --> 04:49:59,880 CONGRESSIONALLY MANDATED 7200 04:49:59,880 --> 04:50:00,160 PROJECTS. 7201 04:50:00,160 --> 04:50:02,840 SO IT USED TO BE THAT CONGRESS 7202 04:50:02,840 --> 04:50:07,240 WOULD INCREASE THE MONEY TO NIH, 7203 04:50:07,240 --> 04:50:08,080 VERY STANDARD FASHION, EACH 7204 04:50:08,080 --> 04:50:10,640 INSTITUTE WOULD GET THE SAME 7205 04:50:10,640 --> 04:50:11,640 INCREASE, MAYBE CANCER WOULD 7206 04:50:11,640 --> 04:50:15,760 PROBABLY GET MORE. 7207 04:50:15,760 --> 04:50:16,960 WHAT HAPPENED AFTER THE 12 YEARS 7208 04:50:16,960 --> 04:50:19,280 OF FLAT FUNDING THAT ENDED IN 7209 04:50:19,280 --> 04:50:21,320 2016, WE DID START TO GET 7210 04:50:21,320 --> 04:50:24,160 INCREASES BUT WE ALSO GOT 7211 04:50:24,160 --> 04:50:27,360 SPECIAL PROGRAMS THAT CONGRESS 7212 04:50:27,360 --> 04:50:28,760 PUT FUNDING INTO AND SO THAT 7213 04:50:28,760 --> 04:50:30,560 WAS -- THAT'S GOOD IF YOU'RE IN 7214 04:50:30,560 --> 04:50:31,960 ONE OF THOSE PROGRAMS. 7215 04:50:31,960 --> 04:50:33,920 FOR US, WE WERE LUCKY BECAUSE 7216 04:50:33,920 --> 04:50:37,520 MOST OF THE PROGRAMS WERE IN 7217 04:50:37,520 --> 04:50:38,520 NEUROLOGICAL DISEASES. 7218 04:50:38,520 --> 04:50:42,040 SO, THE FUNDING FOR DEMENTIA 7219 04:50:42,040 --> 04:50:43,040 RESEARCH INCREASED BY $3 BILLION 7220 04:50:43,040 --> 04:50:46,160 OVER FIVE YEARS. 7221 04:50:46,160 --> 04:50:50,280 WE MANAGE 10% OF THAT MONEY, 7222 04:50:50,280 --> 04:50:52,680 THAT'S NOT SEEN ON THIS TABLE, 7223 04:50:52,680 --> 04:50:53,880 $3 MILLION A YEAR. 7224 04:50:53,880 --> 04:50:56,120 BRAIN INITIATIVE IS A 7225 04:50:56,120 --> 04:50:57,480 MAGNIFICENT PROJECT TO 7226 04:50:57,480 --> 04:50:59,680 UNDERSTAND HOW BRAIN CIRCUITS 7227 04:50:59,680 --> 04:50:59,960 WORK. 7228 04:50:59,960 --> 04:51:01,640 AND JUST MAYBE WE'LL GET INTO 7229 04:51:01,640 --> 04:51:04,240 THAT IN A COUPLE SLIDES DOWN 7230 04:51:04,240 --> 04:51:07,400 FROM NOW, BUT THE PREFACE IS 7231 04:51:07,400 --> 04:51:08,760 ANYONE WITH A NEUROLOGIC 7232 04:51:08,760 --> 04:51:12,000 DISEASE, THE SYMPTOMS ARE DUE TO 7233 04:51:12,000 --> 04:51:13,440 ALTERED CIRCUIT. 7234 04:51:13,440 --> 04:51:15,000 IN THE PAST WE COULD NEVER LOOK 7235 04:51:15,000 --> 04:51:16,640 AT THOSE CIRCUITS AND SEE WHAT 7236 04:51:16,640 --> 04:51:18,600 THEY WERE DOING OR HOW THEY WERE 7237 04:51:18,600 --> 04:51:19,920 NOTE FUNCTIONING. 7238 04:51:19,920 --> 04:51:22,080 BUT NOW THE TECHNOLOGIES ARE 7239 04:51:22,080 --> 04:51:23,280 COMING. 7240 04:51:23,280 --> 04:51:25,080 THAT'S GOING TO REVOLUTIONIZE 7241 04:51:25,080 --> 04:51:27,800 HOW WE DO THERAPEUTIC STUFF. 7242 04:51:27,800 --> 04:51:28,760 PARTICULARLY SOME DIAGNOSTICS. 7243 04:51:28,760 --> 04:51:31,760 THE HEAL INITIATIVE IS A PROGRAM 7244 04:51:31,760 --> 04:51:35,160 IN RESPONSE TO THE OPIOID 7245 04:51:35,160 --> 04:51:36,760 CRISIS. 7246 04:51:36,760 --> 04:51:38,960 AND WE RUN THE PORTION RELATED 7247 04:51:38,960 --> 04:51:40,600 TO DEVELOPING NON-ADDICTIVE PAIN 7248 04:51:40,600 --> 04:51:41,200 THERAPIES. 7249 04:51:41,200 --> 04:51:44,800 NOT ONLY NEW THERAPIES BUT 7250 04:51:44,800 --> 04:51:45,960 BETTER USING REDUCING THERAPIES 7251 04:51:45,960 --> 04:51:50,800 TO REDUCE CHANCE OF ADDICTION, 7252 04:51:50,800 --> 04:51:55,480 ABOUT $280 MILLION. 7253 04:51:55,480 --> 04:51:57,280 AND THEN WE ALSO HAVE TAKEN OVER 7254 04:51:57,280 --> 04:51:59,120 THE MANAGEMENT OF THE 7255 04:51:59,120 --> 04:52:00,320 UNDIAGNOSED DISEASE NETWORK. 7256 04:52:00,320 --> 04:52:02,520 SO THAT WAS RUN OUT OF THE 7257 04:52:02,520 --> 04:52:04,760 COMMON FUND WHICH WAS OFFICE OF 7258 04:52:04,760 --> 04:52:06,560 THE DIRECTOR, DR. COLLINS' 7259 04:52:06,560 --> 04:52:07,160 OFFICE. 7260 04:52:07,160 --> 04:52:08,440 THOSE PROGRAMS RUN FOR TEN 7261 04:52:08,440 --> 04:52:10,600 YEARS, A REALLY GREAT PROGRAM, 7262 04:52:10,600 --> 04:52:12,400 ACROSS THE COUNTRY, WHERE MOSTLY 7263 04:52:12,400 --> 04:52:14,880 KIDS BUT COULD BE ADULTS WHO 7264 04:52:14,880 --> 04:52:17,760 HAVE DISORDERS THAT NO ONE CAN 7265 04:52:17,760 --> 04:52:18,840 DIAGNOSE, THEY CAN APPLY. 7266 04:52:18,840 --> 04:52:20,160 PEOPLE LOOK AT THE RECORDS. 7267 04:52:20,160 --> 04:52:21,440 THEY SEE IF IT LOOKS LIKE 7268 04:52:21,440 --> 04:52:23,440 THERE'S A CHANCE THEY CAN HELP. 7269 04:52:23,440 --> 04:52:26,040 THE ONES THEY CAN, THEY BRING IN 7270 04:52:26,040 --> 04:52:27,480 AND THERE'S A WHOLE TEAM THAT 7271 04:52:27,480 --> 04:52:30,440 FROM AROUND THE COUNTRY INVOLVED 7272 04:52:30,440 --> 04:52:31,400 IN TRYING TO UNDERSTAND WHAT'S 7273 04:52:31,400 --> 04:52:33,600 THE PROBLEM IS. 7274 04:52:33,600 --> 04:52:36,520 THAT'S LED TO A SIGNIFICANT 7275 04:52:36,520 --> 04:52:37,480 PROPORTION OF NEW DIAGNOSES AND 7276 04:52:37,480 --> 04:52:41,360 DISCOVERY OF A NUMBER OF NEW 7277 04:52:41,360 --> 04:52:42,160 GENETIC DISEASES. 7278 04:52:42,160 --> 04:52:44,920 AND I SHOULD HAVE SAID IN THE 7279 04:52:44,920 --> 04:52:48,280 BEGINNING 400 DISEASES WE HAVE, 7280 04:52:48,280 --> 04:52:51,960 THE NUMBER, MAJORITY ARE 7281 04:52:51,960 --> 04:52:52,800 NEUROGENETIC DISEASES. 7282 04:52:52,800 --> 04:52:54,640 AND SO AS YOU'LL SEE WE'RE 7283 04:52:54,640 --> 04:52:57,880 REALLY INTERESTED IN MOVING INTO 7284 04:52:57,880 --> 04:53:02,440 THAT GENE THERAPY SPACE. 7285 04:53:02,440 --> 04:53:07,600 THE BRAIN INITIATIVE, I DID 7286 04:53:07,600 --> 04:53:08,080 MENTION. 7287 04:53:08,080 --> 04:53:10,720 AND I MEAN, I'M THE NEW SURE -- 7288 04:53:10,720 --> 04:53:16,080 IT'S COMPLICATED TO GO THROUGH 7289 04:53:16,080 --> 04:53:19,880 IT ALL BUT JUST AN EXAMPLE, WE 7290 04:53:19,880 --> 04:53:23,720 CAN NOW IN A MOUSE IDENTIFY A 7291 04:53:23,720 --> 04:53:26,120 NEURON GROUP THAT'S RESPONSIBLE 7292 04:53:26,120 --> 04:53:27,240 FOR SOMETHING. 7293 04:53:27,240 --> 04:53:28,320 THEY AIM. 7294 04:53:28,320 --> 04:53:33,560 WE CAN FIND THOSE NEURONS AND 7295 04:53:33,560 --> 04:53:35,960 TURN THEM OFF, ANIMALS HAVE NO 7296 04:53:35,960 --> 04:53:37,920 -- PAIN DOES NOT BOTHER THEM 7297 04:53:37,920 --> 04:53:38,160 ANYMORE. 7298 04:53:38,160 --> 04:53:40,800 THEY WILL STILL FEEL IT BUT IT 7299 04:53:40,800 --> 04:53:42,880 DOESN'T CAUSE ANY SUFFERING. 7300 04:53:42,880 --> 04:53:44,080 SUCH A POWERFUL ABILITY TO DO 7301 04:53:44,080 --> 04:53:47,560 THINGS LIKE THAT. 7302 04:53:47,560 --> 04:53:51,280 YOU CAN DO THAT FOR MANY 7303 04:53:51,280 --> 04:53:58,840 DIFFERENT CORE FEATURES OF 7304 04:53:58,840 --> 04:54:00,680 NEUROLOGY. 7305 04:54:00,680 --> 04:54:03,720 APPETITE, PAIN, DEPRESSION. 7306 04:54:03,720 --> 04:54:04,920 TREMOR, OBSESSIVE COMPULSIVE 7307 04:54:04,920 --> 04:54:06,280 DISORDERS, ALL BEING TARGETED IN 7308 04:54:06,280 --> 04:54:07,040 THAT POSITION. 7309 04:54:07,040 --> 04:54:10,440 THESE ARE JUST IN MICE WHERE YOU 7310 04:54:10,440 --> 04:54:13,720 HAVE THIS CONTROL OVER NEURONS. 7311 04:54:13,720 --> 04:54:14,960 BUT THE IDENTIFICATION OF THE 7312 04:54:14,960 --> 04:54:20,360 CIRCUIT ALLOWS YOU TO DO OTHER 7313 04:54:20,360 --> 04:54:21,000 THINGS USING ELECTRICAL 7314 04:54:21,000 --> 04:54:23,960 STIMULATION, FOR INSTANCE, TO 7315 04:54:23,960 --> 04:54:25,560 TURN OFF PATHWAYS RESPONSIBLE 7316 04:54:25,560 --> 04:54:28,000 FOR THE PATIENT'S SUFFERING. 7317 04:54:28,000 --> 04:54:31,120 IN THE MOUSE, NEW GENE 7318 04:54:31,120 --> 04:54:32,320 THERAPIES, YOU CAN GET THEM, 7319 04:54:32,320 --> 04:54:36,040 TURN THE NEURONS ON AND OFF. 7320 04:54:36,040 --> 04:54:40,080 AND WE TALK ABOUT GENE THERAPY 7321 04:54:40,080 --> 04:54:41,160 FOR RARE NEUROGENIC DISORDERS. 7322 04:54:41,160 --> 04:54:44,800 I THINK IT WAS MENTIONED THIS 7323 04:54:44,800 --> 04:54:46,760 MORNING THAT IF YOU LOOK AT THE 7324 04:54:46,760 --> 04:54:48,320 FUTURE, THIS IS JUST THE 7325 04:54:48,320 --> 04:54:50,320 BEGINNING. 7326 04:54:50,320 --> 04:54:51,920 THESE DISORDERS YOU HEARD ABOUT, 7327 04:54:51,920 --> 04:54:54,000 THESE DISORDERS ARE BEGGING FOR 7328 04:54:54,000 --> 04:54:56,640 THESE KIND OF THERAPIES, BUT 7329 04:54:56,640 --> 04:54:59,800 ONCE THEY ARE SHOWN TO BE SAFE, 7330 04:54:59,800 --> 04:55:01,800 THESE SAME TREATMENTS WILL BE 7331 04:55:01,800 --> 04:55:07,800 USED FOR MORE COMMON DISEASES. 7332 04:55:07,800 --> 04:55:09,720 BUT THAT'S PROBABLY I WOULD SAY 7333 04:55:09,720 --> 04:55:10,640 MAYBE ANOTHER DECADE DOWN THE 7334 04:55:10,640 --> 04:55:12,920 LINE. 7335 04:55:12,920 --> 04:55:18,400 7336 04:55:18,400 --> 04:55:19,920 IN TERMS OF MOVING INTO GENE 7337 04:55:19,920 --> 04:55:23,440 THERAPY YOU HEARD THIS MORNING 7338 04:55:23,440 --> 04:55:24,640 ABOUT BESPOKE GENE THERAPY 7339 04:55:24,640 --> 04:55:26,040 CONSORTIUM THAT WE WORK WITH 7340 04:55:26,040 --> 04:55:27,280 NCATS ON. 7341 04:55:27,280 --> 04:55:30,120 WE ALSO CO-LEAD WITH NCATS 7342 04:55:30,120 --> 04:55:32,200 SOMATIC CELL GENE EDITING 7343 04:55:32,200 --> 04:55:32,520 PROGRAM. 7344 04:55:32,520 --> 04:55:34,800 THE AMP PROGRAM, REMEMBER THAT 7345 04:55:34,800 --> 04:55:39,440 IS TRYING TO WORK WITH THE FDA 7346 04:55:39,440 --> 04:55:41,840 TO DEVELOP A PLATFORM TO USE 7347 04:55:41,840 --> 04:55:43,880 THIS VIRUS, AAV VIRUS, YOU CAN 7348 04:55:43,880 --> 04:55:45,240 PACKAGE THE DIFFERENT GENES? 7349 04:55:45,240 --> 04:55:48,680 AND THE IDEA IS IF YOU CAN BUILD 7350 04:55:48,680 --> 04:55:50,320 THE PLATFORM, YOU CAN JUST 7351 04:55:50,320 --> 04:55:52,920 SWITCH IN THE GENE OF YOUR 7352 04:55:52,920 --> 04:55:54,200 INTEREST, WHATEVER THE DISEASE 7353 04:55:54,200 --> 04:55:59,240 IS, THAT THINGS WILL MOVE MORE 7354 04:55:59,240 --> 04:56:00,800 QUICKLY AND LESS EXPENSIVE. 7355 04:56:00,800 --> 04:56:02,960 THAT'S THE IDEA BEHIND THAT. 7356 04:56:02,960 --> 04:56:06,000 AN INTERESTING PROJECT, 7357 04:56:06,000 --> 04:56:08,720 COMPANIES INVOLVED, ACADEMIA, 7358 04:56:08,720 --> 04:56:14,560 FUNDING ACTUAL CLINICAL TRIALS, 7359 04:56:14,560 --> 04:56:18,680 AND DR. SILVERTHORN IS BUILDING 7360 04:56:18,680 --> 04:56:22,960 A ROAD MAP, FOCUSED ON 7361 04:56:22,960 --> 04:56:23,480 PARTICULAR GENES, GENETIC 7362 04:56:23,480 --> 04:56:24,920 DISORDERS, BUT THE ROAD MAP IS 7363 04:56:24,920 --> 04:56:26,920 GOING TO BE WIDE OPEN, ALLOWING 7364 04:56:26,920 --> 04:56:30,640 OTHER PEOPLE TO FOLLOW IT. 7365 04:56:30,640 --> 04:56:32,920 UNDIAGNOSED DISEASE NETWORK I 7366 04:56:32,920 --> 04:56:33,400 MENTIONED. 7367 04:56:33,400 --> 04:56:37,160 WE DIDN'T TALK ABOUT THE URGENT 7368 04:56:37,160 --> 04:56:39,920 NETWORK, AN NINDS PROGRAM, TO 7369 04:56:39,920 --> 04:56:43,200 DEVELOP GENE THERAPIES, GENOMIC 7370 04:56:43,200 --> 04:56:46,480 THERAPIES, FOR REALLY RARE 7371 04:56:46,480 --> 04:56:47,400 NEUROLOGICKISTAL DISORDERS RUN 7372 04:56:47,400 --> 04:56:48,840 OUT OF NEURONEXT. 7373 04:56:48,840 --> 04:56:51,440 THE IDEA IS THE THE MOVEMENT OF 7374 04:56:51,440 --> 04:56:55,160 GENE THERAPIES AT THE NEURONEXT 7375 04:56:55,160 --> 04:56:56,280 WILL ALLOW THAT NETWORK TO 7376 04:56:56,280 --> 04:56:59,320 BECOME MORE POWERFUL IN TERMS OF 7377 04:56:59,320 --> 04:57:04,360 TYPES OF TREATMENT IT CAN TEST. 7378 04:57:04,360 --> 04:57:13,680 I WANTED TO -- BEFORE I GET OFF 7379 04:57:13,680 --> 04:57:14,320 THESE FUTURE-LOOKING PROJECTS, I 7380 04:57:14,320 --> 04:57:18,680 WANT TO MENTION IN ALL THESE 7381 04:57:18,680 --> 04:57:23,160 AREAS, THE TECHNOLOGIES THAT ARE 7382 04:57:23,160 --> 04:57:24,600 REALLY POWERFUL OFTENTIMES 7383 04:57:24,600 --> 04:57:25,920 REQUIRE BRAIN TISSUE. 7384 04:57:25,920 --> 04:57:28,640 SO, YOU KNOW, WE USED TO LOOK, 7385 04:57:28,640 --> 04:57:31,040 IT WAS ALWAYS IMPORTANT, LOOK 7386 04:57:31,040 --> 04:57:33,680 UNDER MICROSCOPE, WOULD SEE WHAT 7387 04:57:33,680 --> 04:57:36,200 CELLS ARE DOING WHAT, WHAT CELLS 7388 04:57:36,200 --> 04:57:39,360 ARE DYING, WHAT LOOK DIFFERENT. 7389 04:57:39,360 --> 04:57:40,480 NOW TOOLS ALLOW SINGLE CELL 7390 04:57:40,480 --> 04:57:42,440 ANNUAL SILLS OF BRAIN TISSUE, 7391 04:57:42,440 --> 04:57:44,280 YOU CAN GET QUANTITATIVE ABOUT 7392 04:57:44,280 --> 04:57:45,480 WHAT'S MISSING, WHAT GENES HAVE 7393 04:57:45,480 --> 04:57:47,680 BEEN TURNED ON, WHAT THE PROBLEM 7394 04:57:47,680 --> 04:57:48,160 IS. 7395 04:57:48,160 --> 04:57:54,480 YOU CAN SEE CELLS COMPLETELY 7396 04:57:54,480 --> 04:57:56,440 NORMAL, ABOUT TO DIE, A LITTLE 7397 04:57:56,440 --> 04:57:59,880 SICK, OTHERS ARE REALLY SICK, 7398 04:57:59,880 --> 04:58:01,400 NOT AVAILABLE FIVE YEARS AGO. 7399 04:58:01,400 --> 04:58:03,880 THERE'S A HUGE PREMIUM ON BRAIN 7400 04:58:03,880 --> 04:58:06,640 TISSUE FOR ALL THESE DISEASES 7401 04:58:06,640 --> 04:58:06,880 NOW. 7402 04:58:06,880 --> 04:58:09,840 AND A BIG PUSH IS TO APPLY 7403 04:58:09,840 --> 04:58:11,800 SINGLE CELL TECHNOLOGIES TO 7404 04:58:11,800 --> 04:58:12,880 ALMOST EVERY BRAIN DISEASE YOU 7405 04:58:12,880 --> 04:58:14,320 CAN IMAGINE. 7406 04:58:14,320 --> 04:58:16,760 HATS OFF TO TISCH, HELPING US IN 7407 04:58:16,760 --> 04:58:18,200 TERMS OF GETTING BRAIN TISSUE, 7408 04:58:18,200 --> 04:58:19,920 SHE COULD USE YOUR HELP. 7409 04:58:19,920 --> 04:58:21,680 EVERYBODY ELSE IN THE AUDIENCE. 7410 04:58:21,680 --> 04:58:26,400 BECAUSE THIS IS REALLY GETTING 7411 04:58:26,400 --> 04:58:34,080 TO BE A BOTTLE NECK IN GETTING 7412 04:58:34,080 --> 04:58:35,360 THE SCIENCE DONE. 7413 04:58:35,360 --> 04:58:37,200 OKAY. 7414 04:58:37,200 --> 04:58:43,240 7415 04:58:43,240 --> 04:58:48,160 7416 04:58:48,160 --> 04:58:51,800 ONE PROBLEM THAT CAME UP IN THE 7417 04:58:51,800 --> 04:58:53,200 ETHICS SESSION IS THE ISSUE OF 7418 04:58:53,200 --> 04:58:55,480 NEURAL HEALTH EQUITY. 7419 04:58:55,480 --> 04:59:00,000 SO, A LOT OF THINGS PEOPLE ARE 7420 04:59:00,000 --> 04:59:03,360 DOING IS SOPHISTICATED SCIENCE, 7421 04:59:03,360 --> 04:59:06,880 SOPHISTICATED WORK. 7422 04:59:06,880 --> 04:59:10,800 AND IT TENDS TO BE FOCUSED IN 7423 04:59:10,800 --> 04:59:12,560 THESE CERTAIN ACADEMIC 7424 04:59:12,560 --> 04:59:15,400 INSTITUTIONS, CERTAIN CITIES IN 7425 04:59:15,400 --> 04:59:17,320 OUR COUNTRY, BUT THAT LEAVES 98% 7426 04:59:17,320 --> 04:59:20,440 OF THE PEOPLE OUT OF THE BALL 7427 04:59:20,440 --> 04:59:20,760 GAME. 7428 04:59:20,760 --> 04:59:26,880 WE'RE TRYING REALLY HARD TO LOOK 7429 04:59:26,880 --> 04:59:28,560 AT HOW TO REALLY BUILD SPOKES, 7430 04:59:28,560 --> 04:59:32,440 ALL THESE DIFFERENT PROGRAMS WE 7431 04:59:32,440 --> 04:59:34,120 HAVE, SO THERE'S -- MORE PEOPLE 7432 04:59:34,120 --> 04:59:35,000 WILL BE ACCESSIBLE TO THE 7433 04:59:35,000 --> 04:59:36,960 RESEARCH PROGRAM. 7434 04:59:36,960 --> 04:59:39,160 WE HAVE A GOOD OPPORTUNITY IN 7435 04:59:39,160 --> 04:59:40,680 THE ALS, WE'RE ABOUT TO LAUNCH 7436 04:59:40,680 --> 04:59:42,200 IN THAT AREA, HOPEFULLY THE 7437 04:59:42,200 --> 04:59:43,640 OTHER THINGS AS WELL. 7438 04:59:43,640 --> 04:59:47,560 BUT I THINK GOING FOREWARD, 7439 04:59:47,560 --> 04:59:52,080 THERE'S GOING TO BE, YOU KNOW, A 7440 04:59:52,080 --> 04:59:54,160 REAL IMPRIMATUR ON MAKING THESE 7441 04:59:54,160 --> 04:59:55,240 THERAPIES AVAILABLE FOR PEOPLE 7442 04:59:55,240 --> 04:59:57,320 IRREGARDLESS OF WHERE THEY LIVE 7443 04:59:57,320 --> 04:59:59,080 AND WHAT SOCIOECONOMIC STATUS 7444 04:59:59,080 --> 04:59:59,360 IS. 7445 04:59:59,360 --> 05:00:00,040 AND THAT'S PROBABLY NOT 7446 05:00:00,040 --> 05:00:02,800 SOMETHING THAT NIH IS GOING TO 7447 05:00:02,800 --> 05:00:04,520 BE TOO -- WE WON'T HAVE LEVERS 7448 05:00:04,520 --> 05:00:12,080 TO DO MUCH IN THAT SPACE. 7449 05:00:12,080 --> 05:00:14,160 YOU FOLKS THINK ABOUT IT, 7450 05:00:14,160 --> 05:00:15,280 TALKING TO CONGRESS FOLKS, 7451 05:00:15,280 --> 05:00:16,480 GETTING THIS ON THE TABLE IS 7452 05:00:16,480 --> 05:00:19,960 WHAT'S GOING TO MAKE IT HAPPEN. 7453 05:00:19,960 --> 05:00:23,680 AND WE'RE HAPPY TO HELP IN ANY 7454 05:00:23,680 --> 05:00:23,920 WAY. 7455 05:00:23,920 --> 05:00:25,200 BUT I THINK THERE'S GOING TO BE 7456 05:00:25,200 --> 05:00:27,960 A LOT OF ECONOMICS THAT COME 7457 05:00:27,960 --> 05:00:31,120 INTO THIS, AND THERE GETS TO BE 7458 05:00:31,120 --> 05:00:34,440 POLITICS, SO TRYING TO THINK IT 7459 05:00:34,440 --> 05:00:37,600 THROUGH AHEAD OF TIME, SOME OF 7460 05:00:37,600 --> 05:00:38,400 THESE SOPHISTICATED NON-PROFITS 7461 05:00:38,400 --> 05:00:39,240 HAVE DONE THAT. 7462 05:00:39,240 --> 05:00:40,840 BUT WE ALSO HAVE PROBLEMS WITH 7463 05:00:40,840 --> 05:00:45,880 SOME OF OUR COMMON DISEASES, 7464 05:00:45,880 --> 05:00:46,360 EPILEPSY, STROKE, HUGE 7465 05:00:46,360 --> 05:00:48,360 DISPARITIES IN THE WAY PEOPLE 7466 05:00:48,360 --> 05:00:49,560 ARE TREATED. 7467 05:00:49,560 --> 05:00:53,080 AND THEIR RISK FACTORS. 7468 05:00:53,080 --> 05:00:55,720 AND THAT ACTUALLY LEADS TO 7469 05:00:55,720 --> 05:00:56,680 PROBABLY UNAVOIDABLE DEATH 7470 05:00:56,680 --> 05:00:56,920 REALLY. 7471 05:00:56,920 --> 05:00:58,560 SO IN STROKE AREA WE'VE DONE A 7472 05:00:58,560 --> 05:01:02,160 LOT OF WORK IN TERMS OF 7473 05:01:02,160 --> 05:01:03,680 IDENTIFYING IT, NOW WE'RE TRYING 7474 05:01:03,680 --> 05:01:05,800 TO PUT A PROGRAM TOGETHER, 7475 05:01:05,800 --> 05:01:08,280 RICHARD WILL TALK TO YOU 7476 05:01:08,280 --> 05:01:09,440 TOMORROW, WHERE WE DON'T JUST 7477 05:01:09,440 --> 05:01:12,000 WRITE HOW BAD IT IS BUT THROUGH 7478 05:01:12,000 --> 05:01:13,440 AND DO SOMETHING ABOUT IT. 7479 05:01:13,440 --> 05:01:14,880 AND THAT'S REALLY GOING TO BE 7480 05:01:14,880 --> 05:01:20,120 OUR FOCUS GOING FORWARD. 7481 05:01:20,120 --> 05:01:22,280 THERE IS A NEW DATA MANAGEMENT 7482 05:01:22,280 --> 05:01:23,880 AND SHARING POLICY THAT'S COME 7483 05:01:23,880 --> 05:01:27,120 OUT OF THE NIH. 7484 05:01:27,120 --> 05:01:31,600 AND THIS IS RELEVANT TO THE 7485 05:01:31,600 --> 05:01:32,640 GROUP HERE. 7486 05:01:32,640 --> 05:01:35,560 SO, WHAT HAPPENED WAS A BIG PUSH 7487 05:01:35,560 --> 05:01:37,120 IN THE GOVERNMENT, TAXPAYER 7488 05:01:37,120 --> 05:01:39,200 FUNDS THE RESEARCH, THAT DATA 7489 05:01:39,200 --> 05:01:40,480 SHOULD BE AVAILABLE, SHOULDN'T 7490 05:01:40,480 --> 05:01:44,560 BE HIDDEN IN SOMEONE'S DRAWER. 7491 05:01:44,560 --> 05:01:49,040 A NUMBER OF YEARS AGO NIH SAID, 7492 05:01:49,040 --> 05:01:51,040 WELL, STARTING -- TURNS OUT NOW, 7493 05:01:51,040 --> 05:02:01,520 BUT THEY SAID THIS YEARS AGO, 7494 05:02:03,960 --> 05:02:05,200 NIH-FUNDED RESEARCH HAS TO MAKE 7495 05:02:05,200 --> 05:02:08,960 THE DATA AVAILABLE, POST IT 7496 05:02:08,960 --> 05:02:09,200 SOMEWHERE. 7497 05:02:09,200 --> 05:02:13,000 THE OPPORTUNITY FOR YOU TO THINK 7498 05:02:13,000 --> 05:02:14,600 ABOUT HOW TO ORGANIZE THE DATA 7499 05:02:14,600 --> 05:02:15,840 THAT'S HELPFUL TO YOUR GROUP. 7500 05:02:15,840 --> 05:02:20,000 NIH IS NOT DOING THAT. 7501 05:02:20,000 --> 05:02:22,760 THAT PART IS UP TO THE 7502 05:02:22,760 --> 05:02:24,400 SCIENTISTS TO FIGURE OUT HOW TO 7503 05:02:24,400 --> 05:02:25,680 MAKE THE DATA USEFUL. 7504 05:02:25,680 --> 05:02:26,680 GOOD NEWS, WE'RE GOING TO PAY 7505 05:02:26,680 --> 05:02:28,240 THEM TO POST THE DATA. 7506 05:02:28,240 --> 05:02:29,760 BAD NEWS, SOMEBODY HAS TO 7507 05:02:29,760 --> 05:02:32,840 ORGANIZE THEM TO MAKE THE DATA 7508 05:02:32,840 --> 05:02:33,360 USEFUL. 7509 05:02:33,360 --> 05:02:35,120 BUT IT'S AN OPPORTUNITY I THINK 7510 05:02:35,120 --> 05:02:37,200 FOR THE DISEASE ORGANIZATION TO 7511 05:02:37,200 --> 05:02:38,600 TAKE LEADERSHIP ROLE, GET FOLKS 7512 05:02:38,600 --> 05:02:39,840 TOGETHER AND SAY THEY ARE GOING 7513 05:02:39,840 --> 05:02:42,680 TO HAVE TO POST DATA, WHAT 7514 05:02:42,680 --> 05:02:43,960 COMMON DATA ELEMENTS WILL YOU 7515 05:02:43,960 --> 05:02:45,600 USE, HOW ARE WE GOING TO FIND 7516 05:02:45,600 --> 05:02:47,480 IT, CAN YOU TELL US WHERE IT ALL 7517 05:02:47,480 --> 05:02:49,440 IS AND WE'LL HAVE A LIBRARY TO 7518 05:02:49,440 --> 05:02:50,320 GET THROUGH IT. 7519 05:02:50,320 --> 05:02:54,280 TRY AND MAKE THAT DATA USEFUL. 7520 05:02:54,280 --> 05:02:56,680 THAT'S WHERE WE ARE. 7521 05:02:56,680 --> 05:02:57,520 WE'RE INTERESTED IN HELPING BUT 7522 05:02:57,520 --> 05:02:59,080 I DON'T THINK YOU WANT THE 7523 05:02:59,080 --> 05:03:00,200 GOVERNMENT TELLING YOU WHAT WE 7524 05:03:00,200 --> 05:03:01,720 SHOULD DO IN THIS SPACE. 7525 05:03:01,720 --> 05:03:05,760 YOU WANT THE SCIENTISTS AND 7526 05:03:05,760 --> 05:03:06,640 DISEASE ORGANIZATIONS TO MAKE 7527 05:03:06,640 --> 05:03:07,400 THOSE CALLS. 7528 05:03:07,400 --> 05:03:11,240 BUT WE'LL BE PAYING FOR THE 7529 05:03:11,240 --> 05:03:16,280 POSTERS, SO TAKE ADVANTAGE OF 7530 05:03:16,280 --> 05:03:16,520 THAT. 7531 05:03:16,520 --> 05:03:20,800 AND THE LAST SLIDE OF MY GROUP, 7532 05:03:20,800 --> 05:03:25,840 AS YOU KNOW DR. COLLINS STEPPED 7533 05:03:25,840 --> 05:03:29,440 DOWN, SEEMS LIKE 2 1/2 YEARS AGO 7534 05:03:29,440 --> 05:03:30,040 KNOW. 7535 05:03:30,040 --> 05:03:31,520 DR. LARRY TABAK, WHO WAS DEPUTY 7536 05:03:31,520 --> 05:03:33,560 DIRECTOR, HAS BEEN THE ACTING 7537 05:03:33,560 --> 05:03:34,600 SINCE THAT TIME. 7538 05:03:34,600 --> 05:03:37,640 AND IT'S A TOUGH JOB. 7539 05:03:37,640 --> 05:03:40,480 AND THERE WAS A SEARCH. 7540 05:03:40,480 --> 05:03:47,840 AND THE SEARCH DECIDED TO PICK 7541 05:03:47,840 --> 05:03:50,480 ON MONICA BERTAGNOLLI "PICK ON" 7542 05:03:50,480 --> 05:03:52,880 WAS A FREUDIAN SLIP, PICKED 7543 05:03:52,880 --> 05:03:54,400 MONICA BERTAGNOLLI, PICKING ON 7544 05:03:54,400 --> 05:03:56,040 HER BECAUSE IT'S SUCH A TOUGH 7545 05:03:56,040 --> 05:03:59,240 JOB. 7546 05:03:59,240 --> 05:04:00,200 SHE'S THE NCI DIRECTOR 7547 05:04:00,200 --> 05:04:01,840 CURRENTLY, IN THE JOB SEVEN OR 7548 05:04:01,840 --> 05:04:03,960 EIGHT MONTHS, WAITING FOR THE 7549 05:04:03,960 --> 05:04:07,760 SENATE TO KIND OF GET PAST THEIR 7550 05:04:07,760 --> 05:04:08,480 POLITICAL THINGS AND ACTUALLY 7551 05:04:08,480 --> 05:04:10,080 MAKE A DECISION HERE. 7552 05:04:10,080 --> 05:04:15,520 IT'S REALLY IMPORTANT THE NIH 7553 05:04:15,520 --> 05:04:18,160 HAVE A DIRECTOR WHO HAS GOOD 7554 05:04:18,160 --> 05:04:19,040 RELATIONSHIPS WITH THE CONGRESS 7555 05:04:19,040 --> 05:04:21,360 AND I THINK SHE'S GOING TO BE A 7556 05:04:21,360 --> 05:04:24,400 REALLY GOOD. 7557 05:04:24,400 --> 05:04:27,240 SHE'S A SURGEON FROM 7558 05:04:27,240 --> 05:04:27,680 DANA-FARBER. 7559 05:04:27,680 --> 05:04:30,320 SURGEONS TEND TO BE NO NONSENSE 7560 05:04:30,320 --> 05:04:32,240 PEOPLE, GET THINGS DONE. 7561 05:04:32,240 --> 05:04:33,800 THAT WOULD BE FANTASTIC, I 7562 05:04:33,800 --> 05:04:34,000 THINK. 7563 05:04:34,000 --> 05:04:35,560 THAT'S WHAT I WANTED TO LET 7564 05:04:35,560 --> 05:04:38,400 PEOPLE KNOW ABOUT, WHERE WE ARE 7565 05:04:38,400 --> 05:04:41,680 IN THE INSTITUTE. 7566 05:04:41,680 --> 05:04:42,680 WE'RE REALLY EXCITED ABOUT 7567 05:04:42,680 --> 05:04:43,280 THINGS GOING FORWARD. 7568 05:04:43,280 --> 05:04:46,120 WE'RE A LITTLE WORRIED ABOUT THE 7569 05:04:46,120 --> 05:04:46,640 BUDGET. 7570 05:04:46,640 --> 05:04:49,120 WHAT WE'VE BEEN TOLD IS THERE'S 7571 05:04:49,120 --> 05:04:51,560 A REASONABLE CHANCE THAT WE'LL 7572 05:04:51,560 --> 05:04:53,320 HAVE A FLAT BUDGET. 7573 05:04:53,320 --> 05:04:56,040 UNFORTUNATELY THAT'S THE BEST 7574 05:04:56,040 --> 05:04:57,880 NEWS WE COULD GET. 7575 05:04:57,880 --> 05:04:59,000 BUT THAT PUTS US $40 MILLION IN 7576 05:04:59,000 --> 05:05:01,960 THE HOLE, WE'LL HAVE TO CUT 7577 05:05:01,960 --> 05:05:04,440 THINGS TO GET BACK TO THE STABLE 7578 05:05:04,440 --> 05:05:04,640 STATE. 7579 05:05:04,640 --> 05:05:05,880 AND THE REASON IS BECAUSE WHEN 7580 05:05:05,880 --> 05:05:07,840 WE FUND THINGS WE FUND THINGS 7581 05:05:07,840 --> 05:05:09,160 FOR FIVE YEARS. 7582 05:05:09,160 --> 05:05:11,040 SO WE HAVE COMMITMENTS THAT ARE 7583 05:05:11,040 --> 05:05:15,400 GOING OUT, THEY GROW OVER TIME. 7584 05:05:15,400 --> 05:05:18,120 IF WE HAVE TO CUT THAT WOULD BE 7585 05:05:18,120 --> 05:05:18,320 TOUGH. 7586 05:05:18,320 --> 05:05:19,520 EVEN FLAT, A LOT OF THINGS WE 7587 05:05:19,520 --> 05:05:21,720 HOPED TO DO WE WON'T BE ABLE TO 7588 05:05:21,720 --> 05:05:23,520 DO FOR A WHILE. 7589 05:05:23,520 --> 05:05:27,080 KIND OF GO INTO A STEADY STATE 7590 05:05:27,080 --> 05:05:27,280 MODE. 7591 05:05:27,280 --> 05:05:29,800 IF WE HAD A CUT WE WOULD BE IN A 7592 05:05:29,800 --> 05:05:31,480 CRISIS MODE. 7593 05:05:31,480 --> 05:05:34,880 BUT WE HAVE GOTTEN INCREASES 7594 05:05:34,880 --> 05:05:35,400 SINCE 2016. 7595 05:05:35,400 --> 05:05:38,640 SO WE HAVE REALLY GOOD RESOURCES 7596 05:05:38,640 --> 05:05:39,280 GOING FORWARD. 7597 05:05:39,280 --> 05:05:41,640 JUST MIGHT BE A LITTLE BIT OF A 7598 05:05:41,640 --> 05:05:42,640 DIP DEPENDING WHAT CONGRESS DOES 7599 05:05:42,640 --> 05:05:46,040 THIS TIME. 7600 05:05:46,040 --> 05:05:50,280 WE'VE GOT OUR FINGERS CROSSED, 7601 05:05:50,280 --> 05:05:50,800 DEFINITELY APPRECIATE YOU 7602 05:05:50,800 --> 05:05:52,520 TALKING ABOUT THE IMPORTANCE OF 7603 05:05:52,520 --> 05:05:54,720 THIS KIND OF WORK, THAT'S REALLY 7604 05:05:54,720 --> 05:05:57,120 WHAT MAKES IT HAPPEN. 7605 05:05:57,120 --> 05:05:57,520 ALL RIGHT. 7606 05:05:57,520 --> 05:05:58,960 THANKS VERY MUCH. 7607 05:05:58,960 --> 05:05:59,960 I'M HAPPY TO ANSWER ANY 7608 05:05:59,960 --> 05:06:03,960 QUESTIONS. 7609 05:06:03,960 --> 05:06:10,960 7610 05:06:10,960 --> 05:06:11,080 7611 05:06:11,080 --> 05:06:12,920 WE HAVE A LOT OF PROGRAM 7612 05:06:12,920 --> 05:06:14,360 DIRECTORS HERE, PEOPLE WHO DO 7613 05:06:14,360 --> 05:06:16,240 ALL THE WORK, I DO E-MAILS AND 7614 05:06:16,240 --> 05:06:17,840 THEY DO ALL THE WORK. 7615 05:06:17,840 --> 05:06:20,800 AND THEY ARE REALLY FANTASTIC 7616 05:06:20,800 --> 05:06:22,680 PEOPLE, REALLY INVESTED AND LOVE 7617 05:06:22,680 --> 05:06:23,320 WORKING WITH NON-PROFIT 7618 05:06:23,320 --> 05:06:24,960 ORGANIZATIONS. 7619 05:06:24,960 --> 05:06:28,280 >> THANK YOU VERY MUCH. 7620 05:06:28,280 --> 05:06:29,560 CURE SMA, APPRECIATE YOUR 7621 05:06:29,560 --> 05:06:31,520 SUPPORT FOR SMA IN THE PAST AND 7622 05:06:31,520 --> 05:06:33,080 COMMITMENT FOR SMA RESEARCH IN 7623 05:06:33,080 --> 05:06:33,360 THE FUTURE. 7624 05:06:33,360 --> 05:06:36,240 I WANT TO EXPAND UPON YOUR LAST 7625 05:06:36,240 --> 05:06:39,840 COMMENT ABOUT THE NIH BUDGET, 7626 05:06:39,840 --> 05:06:41,800 PARTICULARLY NINDS BUDGET. 7627 05:06:41,800 --> 05:06:43,160 DEBT CEILING DEAL MEANS THAT 7628 05:06:43,160 --> 05:06:46,000 LIKE YOU MENTIONED, LEVEL 7629 05:06:46,000 --> 05:06:47,720 FUNDING IS UNFORTUNATELY A 7630 05:06:47,720 --> 05:06:48,720 VICTORY IN THIS POLITICAL 7631 05:06:48,720 --> 05:06:48,960 CLIMATE. 7632 05:06:48,960 --> 05:06:50,480 WHAT WOULD BE PART OF YOUR 7633 05:06:50,480 --> 05:06:53,280 BUDGET THAT WOULD TAKE THE 7634 05:06:53,280 --> 05:06:55,400 BROADEST HIT IF YOU SAW LEVEL 7635 05:06:55,400 --> 05:06:58,800 FUNDING OR A CUT BEING PROPOSED 7636 05:06:58,800 --> 05:07:00,760 IN THE HOUSE BILL? 7637 05:07:00,760 --> 05:07:01,200 >> RIGHT. 7638 05:07:01,200 --> 05:07:04,080 WELL, WHAT WE HAVE NOW IS WE 7639 05:07:04,080 --> 05:07:06,160 HAVE GROUPS, I MENTIONED THREE 7640 05:07:06,160 --> 05:07:07,720 DIVISIONS. 7641 05:07:07,720 --> 05:07:09,120 THOSE THREE DIVISIONS ARE NOW 7642 05:07:09,120 --> 05:07:15,800 PLANNING ON HOW TO ABSORB FLAT, 7643 05:07:15,800 --> 05:07:17,640 WHICH MEANS $40 MILLION DEFICIT, 7644 05:07:17,640 --> 05:07:18,280 OR A CUT. 7645 05:07:18,280 --> 05:07:22,560 THEY WILL COME UP WITH 7646 05:07:22,560 --> 05:07:22,920 RECOMMENDATIONS. 7647 05:07:22,920 --> 05:07:24,960 GENERALLY WE -- IT'S OUR 7648 05:07:24,960 --> 05:07:27,360 RESPONSIBILITY TO OPERATE IN A 7649 05:07:27,360 --> 05:07:28,040 FLAT SITUATION. 7650 05:07:28,040 --> 05:07:30,680 IF THE NIH GETS A CUT, I THINK 7651 05:07:30,680 --> 05:07:33,400 WHAT WE PROBABLY HAVE TO DO IS 7652 05:07:33,400 --> 05:07:35,360 PASS THAT CUT TO ALL THE GRANTS 7653 05:07:35,360 --> 05:07:37,320 THAT ARE ACTUALLY OUT THERE AS 7654 05:07:37,320 --> 05:07:41,280 WELL AS NEW GRANTS. 7655 05:07:41,280 --> 05:07:42,800 IF WE ARE FLAT, WE'LL PROBABLY 7656 05:07:42,800 --> 05:07:44,120 TRY TO ABSORB BY DECREASING 7657 05:07:44,120 --> 05:07:46,840 AMOUNT OF MONEY THAT'S GOING TO 7658 05:07:46,840 --> 05:07:47,760 THE NEW GRANTS. 7659 05:07:47,760 --> 05:07:54,680 THEN WE HAVE THIS ISSUE OF, YOU 7660 05:07:54,680 --> 05:07:56,240 KNOW, FOR INSTANCE THERE ARE 7661 05:07:56,240 --> 05:07:59,120 SOME PEOPLE WHO IF THEY ARE NOT 7662 05:07:59,120 --> 05:08:02,080 FUNDED, SAY YOUNG INVESTIGATOR 7663 05:08:02,080 --> 05:08:03,080 DOESN'T GET THIS FUNDING, THEY 7664 05:08:03,080 --> 05:08:04,480 WILL LEAVE AND WON'T COME BACK. 7665 05:08:04,480 --> 05:08:07,000 THEY WILL BE GONE FOREVER. 7666 05:08:07,000 --> 05:08:10,720 SO WHAT WE'LL TRY AND PUT 7667 05:08:10,720 --> 05:08:14,320 EMPHASIS ON, THE YOUNG 7668 05:08:14,320 --> 05:08:15,120 INVESTIGATORS, EARLY-STAGE 7669 05:08:15,120 --> 05:08:16,960 INVESTIGATORS, ALSO PEOPLE WHO 7670 05:08:16,960 --> 05:08:18,600 HAVE ONLY ONE NIH GRANT, AND 7671 05:08:18,600 --> 05:08:20,920 THEY COME REALLY CLOSE TO 7672 05:08:20,920 --> 05:08:22,440 GETTING FUNDED BUT THEY DON'T 7673 05:08:22,440 --> 05:08:23,640 MAKE THE PAYLINE. 7674 05:08:23,640 --> 05:08:26,240 IF THEY DON'T GET A GRANT THEIR 7675 05:08:26,240 --> 05:08:28,000 LABS WILL CLOSE AND THEY WILL 7676 05:08:28,000 --> 05:08:30,960 LOSE THEIR JOB IN THE LAB, WE'LL 7677 05:08:30,960 --> 05:08:38,080 BASICALLY LOOK AT THAT GROUP. 7678 05:08:38,080 --> 05:08:41,920 I HATE TO SAY THIS, WE HAVE $72 7679 05:08:41,920 --> 05:08:43,120 MULTI-CLINICAL TRIAL WE'D LIKE 7680 05:08:43,120 --> 05:08:45,840 TO DO, THAT WOULD BE HARD TO DO 7681 05:08:45,840 --> 05:08:48,280 IF WE GOT FLAT BUDGET. 7682 05:08:48,280 --> 05:08:51,400 WE MIGHT HAVE TO PUT THAT OFF 7683 05:08:51,400 --> 05:08:52,320 UNTIL THE FINANCIAL SITUATION 7684 05:08:52,320 --> 05:08:55,040 LOOKS BETTER. 7685 05:08:55,040 --> 05:08:58,360 7686 05:08:58,360 --> 05:08:59,560 THOSE ARE HARD DECISIONS. 7687 05:08:59,560 --> 05:09:02,480 WE WANT TO ACTUALLY KEEP THE 7688 05:09:02,480 --> 05:09:06,080 WHOLE ECOSYSTEM HEALTHY AND ALL 7689 05:09:06,080 --> 05:09:07,000 THE DIFFERENT ASPECTS, AND 7690 05:09:07,000 --> 05:09:09,440 BECAUSE THESE THINGS GO UP AND 7691 05:09:09,440 --> 05:09:09,640 DOWN. 7692 05:09:09,640 --> 05:09:11,280 YOU DON'T WANT TO DO, IN THE 7693 05:09:11,280 --> 05:09:12,800 DOWN SIDE, DO SOMETHING THAT'S 7694 05:09:12,800 --> 05:09:17,400 GOING TO HURT YOU FOR TEN YEARS. 7695 05:09:17,400 --> 05:09:17,840 7696 05:09:17,840 --> 05:09:19,920 >> HI, WALTER, DIANA GRAY, I'M 7697 05:09:19,920 --> 05:09:24,640 THE CEO OF THE HYDROCEPHALUS 7698 05:09:24,640 --> 05:09:25,840 ASSOCIATION. 7699 05:09:25,840 --> 05:09:26,200 THANK YOU. 7700 05:09:26,200 --> 05:09:27,200 I'M RIGHT HERE. 7701 05:09:27,200 --> 05:09:30,480 THANK YOU FOR THE PRESENTATION. 7702 05:09:30,480 --> 05:09:33,200 IF HAD YOU ALL THE MONEY COULD 7703 05:09:33,200 --> 05:09:34,840 YOU IMAGINE FOR THE NINDS, WHAT 7704 05:09:34,840 --> 05:09:37,040 WOULD YOU DO, WHAT ARE YOU 7705 05:09:37,040 --> 05:09:38,600 MISSING, WHAT DO YOU LAY AT WAKE 7706 05:09:38,600 --> 05:09:40,560 AND NIGHT AND THINK IF I COULD 7707 05:09:40,560 --> 05:09:42,080 JUST HAVE THIS MUCH MORE I COULD 7708 05:09:42,080 --> 05:09:45,520 DO X, WHAT WOULD IT BE? 7709 05:09:45,520 --> 05:09:50,960 7710 05:09:50,960 --> 05:09:55,000 >> BEFORE I GO TO THE MONEY 7711 05:09:55,000 --> 05:10:00,600 PART, I'D SAY THAT THE PEOPLE 7712 05:10:00,600 --> 05:10:02,920 WHO MAKE IT HAPPEN, SO NOT 7713 05:10:02,920 --> 05:10:05,840 NECESSARILY -- YOU DON'T REALLY 7714 05:10:05,840 --> 05:10:06,840 BUY THE PEOPLE. 7715 05:10:06,840 --> 05:10:09,560 SOMETHING ELSE HAS TO HAPPEN TO 7716 05:10:09,560 --> 05:10:12,960 GET THE PEOPLE TO COMMIT TO THIS 7717 05:10:12,960 --> 05:10:20,920 KIND OF CAREER. 7718 05:10:20,920 --> 05:10:22,280 IT'S OUR JOB TO PURSUE. 7719 05:10:22,280 --> 05:10:25,000 IN THAT SPACE WHAT WE KNOW IS AS 7720 05:10:25,000 --> 05:10:27,640 I MENTIONED HOW WE FUND THINGS 7721 05:10:27,640 --> 05:10:29,320 IS WE FUND THE BEST SCORES UNTIL 7722 05:10:29,320 --> 05:10:32,040 WE RUN OUT OF MONEY, AND THERE 7723 05:10:32,040 --> 05:10:35,200 ARE CERTAIN AREAS WHERE, SAY, 7724 05:10:35,200 --> 05:10:36,560 FOR INSTANCE, THE EARLY-STAGE 7725 05:10:36,560 --> 05:10:39,280 INVESTIGATORS, WHERE WE MADE A 7726 05:10:39,280 --> 05:10:40,400 COMMITMENT THAT ALTHOUGH WE RUN 7727 05:10:40,400 --> 05:10:45,640 OUT OF MONEY AT THE 14th 7728 05:10:45,640 --> 05:10:47,080 PERCENTILE GRANT WE'LL FUND 7729 05:10:47,080 --> 05:10:47,680 EARLY-STAGE INVESTIGATORS TO 25 7730 05:10:47,680 --> 05:10:49,880 BECAUSE WE REALLY HAVE TO INVEST 7731 05:10:49,880 --> 05:10:50,680 IN THE EARLY-STAGE 7732 05:10:50,680 --> 05:10:51,520 INVESTIGATORS. 7733 05:10:51,520 --> 05:10:53,600 SO THEN WE DID ANALYSIS TO SEE, 7734 05:10:53,600 --> 05:10:55,360 WELL, IS THERE ANY DIFFERENCE 7735 05:10:55,360 --> 05:10:59,960 BETWEEN PEOPLE WHO GOT BELOW 14 7736 05:10:59,960 --> 05:11:00,240 VERSUS 23? 7737 05:11:00,240 --> 05:11:01,040 NO DIFFERENCE. 7738 05:11:01,040 --> 05:11:03,360 AS A MATTER OF FACT, THE PEOPLE 7739 05:11:03,360 --> 05:11:07,400 WHO GOT THE GRANT EARLY, THEY 7740 05:11:07,400 --> 05:11:08,720 ARE MAYBE BIG HEAD AND DON'T DO 7741 05:11:08,720 --> 05:11:11,120 AS WELL IN THE LONG RUN. 7742 05:11:11,120 --> 05:11:13,760 SOME OF THE REAL, YOU KNOW, SOME 7743 05:11:13,760 --> 05:11:17,280 OF THE PEOPLE WHO REALLY WORK 7744 05:11:17,280 --> 05:11:18,920 HARD AND KEEP PERSISTING, THEY 7745 05:11:18,920 --> 05:11:19,840 SOMETIMES DO BETTER. 7746 05:11:19,840 --> 05:11:23,320 SO WHAT WE KNOW EVERY TIME WE'VE 7747 05:11:23,320 --> 05:11:25,520 LOOKED, THAT ABOVE WHERE WE FUND 7748 05:11:25,520 --> 05:11:31,240 PEOPLE ARE JUST AS GOOD AS THE 7749 05:11:31,240 --> 05:11:32,880 PEOPLE WHO GOT IN RIGHT UNDER 7750 05:11:32,880 --> 05:11:33,920 THE FUNDING. 7751 05:11:33,920 --> 05:11:35,520 WE LEAVE A LOT OF GOOD RESEARCH 7752 05:11:35,520 --> 05:11:38,560 ON THE TABLE, THAT'S FOR SURE. 7753 05:11:38,560 --> 05:11:41,760 AND THEN WE HAVE BEEN ABLE TO DO 7754 05:11:41,760 --> 05:11:43,200 CERTAIN THINGS FOR CERTAIN 7755 05:11:43,200 --> 05:11:45,920 REASONS THAT HAPPEN TO US. 7756 05:11:45,920 --> 05:11:48,520 SO WE HAVE LIKE THE BRAIN 7757 05:11:48,520 --> 05:11:51,600 INITIATIVE, WE HAVE HEAL 7758 05:11:51,600 --> 05:11:55,960 INITIATIVE, ALS PROGRAM. 7759 05:11:55,960 --> 05:11:57,520 WE COULD -- THEY ARE REALLY GOOD 7760 05:11:57,520 --> 05:11:58,880 PROGRAMS, WE COULD DO THOSE IN 7761 05:11:58,880 --> 05:12:01,160 OTHER DISEASES IF WE HAD THE 7762 05:12:01,160 --> 05:12:02,560 MONEY. 7763 05:12:02,560 --> 05:12:04,440 I THINK BUILDING THE 7764 05:12:04,440 --> 05:12:05,520 PARTNERSHIPS HAVE BEEN REALLY 7765 05:12:05,520 --> 05:12:07,800 THINK A POWERFUL TOOL FOR US TO 7766 05:12:07,800 --> 05:12:09,160 BUILD PARTNERSHIPS WITH THE 7767 05:12:09,160 --> 05:12:10,760 NON-PROFITS AND THE INDUSTRY 7768 05:12:10,760 --> 05:12:13,400 AROUND A CERTAIN AREA. 7769 05:12:13,400 --> 05:12:15,280 WE HAVE ONE IN PARKINSON'S, 7770 05:12:15,280 --> 05:12:16,160 ACCELERATED MEDICINE 7771 05:12:16,160 --> 05:12:16,480 PARTNERSHIP. 7772 05:12:16,480 --> 05:12:21,840 NOW WE'RE BUILDING ONE IN ALS. 7773 05:12:21,840 --> 05:12:24,160 AND THEN WE HAVE, YOU KNOW, 7774 05:12:24,160 --> 05:12:26,120 NETWORKS THAT HAVE BEEN REALLY 7775 05:12:26,120 --> 05:12:27,680 QUITE PRODUCTIVE THAT WE THINK 7776 05:12:27,680 --> 05:12:30,160 WE COULD GET MORE THERAPIES IN. 7777 05:12:30,160 --> 05:12:32,720 WE COULD REACH OUT TO BIOTECH 7778 05:12:32,720 --> 05:12:34,120 COMPANIES MORE, TRY AND PULL 7779 05:12:34,120 --> 05:12:36,120 THEM IN, PARTICULARLY IN THE 7780 05:12:36,120 --> 05:12:38,600 DISEASES THAT ARE SEEN AS HIGH 7781 05:12:38,600 --> 05:12:39,040 RISK. 7782 05:12:39,040 --> 05:12:41,000 WE COULD DO MORE THERE. 7783 05:12:41,000 --> 05:12:42,960 THE SMALL BUSINESS GRANTS, WHEN 7784 05:12:42,960 --> 05:12:44,240 I CAME, YOU KNOW, SMALL BUSINESS 7785 05:12:44,240 --> 05:12:47,600 GRANT WAS AN EASY WAY TO GET 7786 05:12:47,600 --> 05:12:49,360 MONEY. 7787 05:12:49,360 --> 05:12:50,760 IT WASN'T THAT COMPETITIVE. 7788 05:12:50,760 --> 05:12:51,960 NOW SMALL BUSINESS GRANTS ARE 7789 05:12:51,960 --> 05:12:54,720 REALLY GOOD NOW. 7790 05:12:54,720 --> 05:12:58,440 SO, AGAIN, I THINK OUR FIELD IS 7791 05:12:58,440 --> 05:13:02,560 GROWING AND MUCH MORE WE CAN DO. 7792 05:13:02,560 --> 05:13:04,320 THERE'S NOT ONE THING I WOULD 7793 05:13:04,320 --> 05:13:07,400 SAY WE WOULD DO WITH THE MONEY 7794 05:13:07,400 --> 05:13:09,120 BUT I THINK WE -- YOU KNOW, WHAT 7795 05:13:09,120 --> 05:13:13,680 WE USED TO SAY, REALLY TRUE, 7796 05:13:13,680 --> 05:13:15,200 THINGS ARE GOING WELL, THEY 7797 05:13:15,200 --> 05:13:16,600 COULD GO FASTER IF WE HAD MORE 7798 05:13:16,600 --> 05:13:20,000 MONEY. 7799 05:13:20,000 --> 05:13:24,920 7800 05:13:24,920 --> 05:13:30,400 7801 05:13:30,400 --> 05:13:31,480 7802 05:13:31,480 --> 05:13:33,680 >> IT'S AN EASY ONE. 7803 05:13:33,680 --> 05:13:35,440 I'M STILL STRUCK BY THE JUST 7804 05:13:35,440 --> 05:13:38,480 HOPE FOR THE WHOLE NEUROLOGICAL 7805 05:13:38,480 --> 05:13:38,880 SPACE. 7806 05:13:38,880 --> 05:13:44,160 FROM WHERE IT WAS 10 TO 20 YEARS 7807 05:13:44,160 --> 05:13:46,320 AGO, 90% FAILURE RATES IN 7808 05:13:46,320 --> 05:13:48,120 CLINICAL TRIALS, THERE'S A LONG 7809 05:13:48,120 --> 05:13:51,720 ROAD AHEAD BUT I'M INCREDIBLY 7810 05:13:51,720 --> 05:13:55,200 HOPEFUL. 7811 05:13:55,200 --> 05:13:59,120 YOU'VE GOT 400 DISEASES IN YOUR 7812 05:13:59,120 --> 05:14:00,520 PORTFOLIO, THE QUESTION OF WHO 7813 05:14:00,520 --> 05:14:02,560 IS GOING TO PAY FOR THOSE DRUGS 7814 05:14:02,560 --> 05:14:04,840 IS NOT YOURS TO ANSWER BUT 7815 05:14:04,840 --> 05:14:07,040 REFLECT FROM YOUR EXPERIENCE AND 7816 05:14:07,040 --> 05:14:09,320 POSITION, IT FEELS LIKE AS WE DO 7817 05:14:09,320 --> 05:14:11,400 GET SUCCESS FOR ALL THE RARE 7818 05:14:11,400 --> 05:14:12,720 DISEASES WHICH ARE GOING TO CHIP 7819 05:14:12,720 --> 05:14:15,320 AWAY MORE AND MORE AS WELL AS 7820 05:14:15,320 --> 05:14:16,880 MASSIVE ONES LIKE ALZHEIMER'S, 7821 05:14:16,880 --> 05:14:19,280 THAT ARE NOT RARE BUT HAVE 7822 05:14:19,280 --> 05:14:21,160 PREVIOUSLY NOT REALLY BEEN PAID 7823 05:14:21,160 --> 05:14:24,480 FOR, WE NEED TO RESET HOW WE PAY 7824 05:14:24,480 --> 05:14:24,800 FOR ALL DRUGS. 7825 05:14:24,800 --> 05:14:26,560 I DON'T KNOW THE ANSWERS TO THAT 7826 05:14:26,560 --> 05:14:30,680 BUT I'D LOVE TO THROW IT AT YOU 7827 05:14:30,680 --> 05:14:33,240 AND HEAR YOUR WORDS OF WISDOM 7828 05:14:33,240 --> 05:14:34,040 FOR A FEW MINUTES. 7829 05:14:34,040 --> 05:14:34,520 >> RIGHT. 7830 05:14:34,520 --> 05:14:39,560 THE WAY I THINK ABOUT IT IS THAT 7831 05:14:39,560 --> 05:14:42,200 THE GREAT THING ABOUT OUR JOB IS 7832 05:14:42,200 --> 05:14:44,480 THAT WE TAKE PROBLEMS THAT HAVE 7833 05:14:44,480 --> 05:14:47,520 NO SOLUTIONS AND TRY TO GET A 7834 05:14:47,520 --> 05:14:49,160 SOLUTION FOR IT. 7835 05:14:49,160 --> 05:14:52,240 AND THEN THE QUESTION COMES, 7836 05:14:52,240 --> 05:14:53,920 WHAT IS THE -- WE THROW IT OVER 7837 05:14:53,920 --> 05:14:54,640 THE FENCE 7838 05:14:54,640 --> 05:14:56,160 WHAT IS THE COMMUNITY GOING TO 7839 05:14:56,160 --> 05:14:56,960 DO ABOUT IT? 7840 05:14:56,960 --> 05:15:02,400 SOMETIMES WE CAN HELP IN THAT 7841 05:15:02,400 --> 05:15:02,720 SPACE. 7842 05:15:02,720 --> 05:15:05,160 AND -- BUT THE DISEASES THAT 7843 05:15:05,160 --> 05:15:07,120 WE'RE TALKING ABOUT ARE NOT THE 7844 05:15:07,120 --> 05:15:11,080 KIND OF THINGS PEOPLE CAN 7845 05:15:11,080 --> 05:15:11,840 IGNORE. 7846 05:15:11,840 --> 05:15:12,520 SO I'M VERY CONFIDENT THAT I 7847 05:15:12,520 --> 05:15:14,280 DON'T KNOW THE ANSWER BUT I 7848 05:15:14,280 --> 05:15:16,040 THINK PEOPLE WILL FIND A WAY TO 7849 05:15:16,040 --> 05:15:19,720 DO ANY A LESS EXPENSIVE WAY, TO 7850 05:15:19,720 --> 05:15:20,720 MAKE IT MORE ACCESSIBLE, BECAUSE 7851 05:15:20,720 --> 05:15:23,440 THIS IS JUST NOT RIGHT THAT IT'S 7852 05:15:23,440 --> 05:15:25,760 NOT DONE. 7853 05:15:25,760 --> 05:15:31,560 AND MY EXPERIENCE, I WORKED IN 7854 05:15:31,560 --> 05:15:31,880 HUNTINGTON'S. 7855 05:15:31,880 --> 05:15:34,000 AND WE HAD A LOT -- WE FOUND THE 7856 05:15:34,000 --> 05:15:35,600 GENE, THOUGHT WE WOULD HAVE A 7857 05:15:35,600 --> 05:15:37,400 TREATMENT IN TEN YEARS, IT'S NOW 7858 05:15:37,400 --> 05:15:38,680 25 YEARS, AND NO TREATMENT. 7859 05:15:38,680 --> 05:15:46,120 BUT THEN I WORKED IN STROKE, 7860 05:15:46,120 --> 05:15:47,560 BASICALLY WORKED AT TAKING CLOTS 7861 05:15:47,560 --> 05:15:48,760 OUT OF PEOPLE WITH ACUTE STROKE, 7862 05:15:48,760 --> 05:15:53,120 IT WAS LIKE A MIRACLE. 7863 05:15:53,120 --> 05:15:56,440 NOW WE HAVE, YOU KNOW, NOW IT'S 7864 05:15:56,440 --> 05:15:58,400 GONE ACROSS THE COUNTRY. 7865 05:15:58,400 --> 05:16:00,360 WHEN I STARTED, YOU COULDN'T GET 7866 05:16:00,360 --> 05:16:02,840 ANYBODY TO TAKE A STROKE PATIENT 7867 05:16:02,840 --> 05:16:06,280 ON THE TEAM BECAUSE IT WAS SUCH 7868 05:16:06,280 --> 05:16:06,920 A TERRIBLE OUTCOME. 7869 05:16:06,920 --> 05:16:08,360 YOU HAVE ONE OF THESE LARGE 7870 05:16:08,360 --> 05:16:09,440 CLOTS, YOU'RE DEAD. 7871 05:16:09,440 --> 05:16:11,000 NOW IT'S COMPLETELY CHANGED. 7872 05:16:11,000 --> 05:16:14,840 THE WHOLE SYSTEM IS CHANGED. 7873 05:16:14,840 --> 05:16:16,120 FROM MY EXPERIENCE, THERE'S 7874 05:16:16,120 --> 05:16:18,080 NOTHING LIKE AN EFFECTIVE DRUG 7875 05:16:18,080 --> 05:16:20,400 TO ACTUALLY CHANGE THE HEALTH 7876 05:16:20,400 --> 05:16:20,880 CARE SYSTEM. 7877 05:16:20,880 --> 05:16:23,120 YOU CAN DO EVERYTHING YOU WANT, 7878 05:16:23,120 --> 05:16:24,560 IF THERE'S NO TREATMENT, IT 7879 05:16:24,560 --> 05:16:27,080 SEEMS LIKE THE YOU CAN ONLY MAKE 7880 05:16:27,080 --> 05:16:27,520 INCREMENTAL CHANGES. 7881 05:16:27,520 --> 05:16:31,240 ONCE YOU HAVE A NEW TREATMENT IT 7882 05:16:31,240 --> 05:16:33,400 FORCES CHANGE AND USUALLY -- IN 7883 05:16:33,400 --> 05:16:34,520 OUR COUNTRY STROKE, IT WORKED 7884 05:16:34,520 --> 05:16:36,920 OUT FOR THE PEOPLE IN THE 7885 05:16:36,920 --> 05:16:37,160 COUNTRY. 7886 05:16:37,160 --> 05:16:38,120 NOT EVERYBODY. 7887 05:16:38,120 --> 05:16:40,840 IT'S STILL NOT EQUALLY 7888 05:16:40,840 --> 05:16:41,160 ACCESSIBLE. 7889 05:16:41,160 --> 05:16:43,680 AND THAT WAS A BIG THING. 7890 05:16:43,680 --> 05:16:45,440 HAD YOU TO TREAT PEOPLE WITHIN 7891 05:16:45,440 --> 05:16:46,640 THREE HOURS OF A STROKE. 7892 05:16:46,640 --> 05:16:48,840 THERE'S NO WAY THAT COULD EVER 7893 05:16:48,840 --> 05:16:51,840 HAPPEN BUT NOW IT'S ROUTINE. 7894 05:16:51,840 --> 05:16:54,560 SO I THINK IF YOU GET SOMETHING 7895 05:16:54,560 --> 05:16:56,960 THAT WORKS, THE SYSTEM WILL 7896 05:16:56,960 --> 05:17:00,560 FIGURE OUT ITS WAY AREN'T. 7897 05:17:00,560 --> 05:17:03,200 NOW, THE MONEY SIDE, THAT'S 7898 05:17:03,200 --> 05:17:04,080 DIFFERENT. 7899 05:17:04,080 --> 05:17:08,640 IT'S NOT JUST US. 7900 05:17:08,640 --> 05:17:11,400 IT'S THE CANCER DRUGS, 7901 05:17:11,400 --> 05:17:13,480 RHEUMATOLOGY DRUGS, BIOLOGICS, 7902 05:17:13,480 --> 05:17:17,000 AND, YOU KNOW, THINK THAT'S 7903 05:17:17,000 --> 05:17:18,960 GOING TO REQUIRE A CHANGE IN THE 7904 05:17:18,960 --> 05:17:23,040 WAY THE COUNTRY LOOKS AT THINGS. 7905 05:17:23,040 --> 05:17:25,440 I DON'T THINK THEY ARE GOING TO 7906 05:17:25,440 --> 05:17:29,400 LEAVE PEOPLE UNTREATED. 7907 05:17:29,400 --> 05:17:33,280 7908 05:17:33,280 --> 05:17:35,160 >> I HAVE A QUESTION. 7909 05:17:35,160 --> 05:17:37,880 MORE OF A COMMENT. 7910 05:17:37,880 --> 05:17:47,720 BRANDY, CO-FOUNDER OF SUPPORT 7911 05:17:47,720 --> 05:17:48,400 FIBROMYALGIA, ESTABLISHED 7912 05:17:48,400 --> 05:17:49,040 SHORTLY AFTER BEING DIAGNOSED, 7913 05:17:49,040 --> 05:17:51,560 MY MOM IN THE '90s WHEN IT WAS 7914 05:17:51,560 --> 05:17:53,000 COMING INTO THE MEDICAL FIELD. 7915 05:17:53,000 --> 05:17:54,840 ONE OF THE DISAPPOINTMENTS I HAD 7916 05:17:54,840 --> 05:17:56,840 WAS THERE WAS NOT MUCH OF A 7917 05:17:56,840 --> 05:17:57,920 DIFFERENCE BETWEEN THE TREATMENT 7918 05:17:57,920 --> 05:17:59,120 METHODS THAT MY MOTHER RECEIVED 7919 05:17:59,120 --> 05:18:03,840 AND IN '90s AND WHAT I 7920 05:18:03,840 --> 05:18:06,120 RECEIVED IN 2012. 7921 05:18:06,120 --> 05:18:09,200 WE CREATED THE NON-PROFIT 7922 05:18:09,200 --> 05:18:12,400 FIBROMYALGIA TO HOPE OF GETTING 7923 05:18:12,400 --> 05:18:13,480 MORE RESEARCH FOR FIBROMYALGIA 7924 05:18:13,480 --> 05:18:15,000 AND GIVE THE COMMUNITY HOPE 7925 05:18:15,000 --> 05:18:16,960 THERE ARE PEOPLE FIGHTING TO GET 7926 05:18:16,960 --> 05:18:18,520 ADVANCEMENTS IN TREATMENT AND 7927 05:18:18,520 --> 05:18:20,600 HELPING OUR PROVIDERS. 7928 05:18:20,600 --> 05:18:23,560 SO THIS IS MORE OF A COMMENT TO 7929 05:18:23,560 --> 05:18:24,200 SAY WE APPRECIATE THIS 7930 05:18:24,200 --> 05:18:25,920 OPPORTUNITY TO BE ABLE TO 7931 05:18:25,920 --> 05:18:27,480 CONNECT WITH RESEARCHERS AND 7932 05:18:27,480 --> 05:18:28,560 CONNECT WITH OTHER NON-PROFITS 7933 05:18:28,560 --> 05:18:32,040 TO LEARN HOW WE CAN FURTHER 7934 05:18:32,040 --> 05:18:32,480 THOSE EFFORTS. 7935 05:18:32,480 --> 05:18:33,840 THAT'S WHAT I JUST WANTED TO 7936 05:18:33,840 --> 05:18:36,240 SAY. 7937 05:18:36,240 --> 05:18:38,400 7938 05:18:38,400 --> 05:18:43,520 >> I REALLY APPRECIATE THAT. 7939 05:18:43,520 --> 05:18:45,400 AND CERTAINLY WE ARE NOW LEADING 7940 05:18:45,400 --> 05:18:53,520 THE PAIN RESEARCH FOR THE HEAL 7941 05:18:53,520 --> 05:18:55,800 INITIATIVE, AND TRYING TO BUILD 7942 05:18:55,800 --> 05:18:57,680 FROM LOW LEVEL A STRONG PAIN 7943 05:18:57,680 --> 05:19:02,720 RESEARCH STRUCTURE AND HOPE IT 7944 05:19:02,720 --> 05:19:07,960 CAN HELP WITH YOU FIBROMYALGIA. 7945 05:19:07,960 --> 05:19:10,160 7946 05:19:10,160 --> 05:19:10,360 OKAY. 7947 05:19:10,360 --> 05:19:11,120 THINK WE'RE GOOD. 7948 05:19:11,120 --> 05:19:12,680 IT WAS A REALLY GREAT DAY, I 7949 05:19:12,680 --> 05:19:15,520 MUST SAY. 7950 05:19:15,520 --> 05:19:21,120 7951 05:19:21,120 --> 05:19:22,400 >> THANK YOU, WALTER. 7952 05:19:22,400 --> 05:19:23,600 WE HAVE POSTERS. 7953 05:19:23,600 --> 05:19:27,760 WE HAVE DIVISION DIRECTORS. 7954 05:19:27,760 --> 05:19:30,800 WE HAVE LEADERSHIP AND PROGRAM 7955 05:19:30,800 --> 05:19:32,880 DIRECTORS TO TALK WITH YOU. 7956 05:19:32,880 --> 05:19:34,680 WE CAN CONTINUE THE MEETING, 7957 05:19:34,680 --> 05:19:36,960 CONTINUE THE CONVERSATION IN THE 7958 05:19:36,960 --> 05:19:37,760 POSTER SPACE. 7959 05:19:37,760 --> 05:19:39,360 TOMORROW AT 10:00 WE'LL START 7960 05:19:39,360 --> 05:19:42,160 PROMPTLY AT 10:00 TOMORROW. 7961 05:19:42,160 --> 05:19:42,760 >> GREAT. 7962 05:19:42,760 --> 05:19:43,400 >> THANK YOU. 7963 05:19:43,400 --> 00:00:00,000 >> THANKS, EVERYONE.