1 00:00:05,040 --> 00:04:12,000 >>WELCOME TO NIH ALS WORKSHOP 2 00:04:12,000 --> 00:04:13,840 HAS HELPED UNDERSTAND THE 3 00:04:13,840 --> 00:04:21,040 BIOLOGY AS WELL. AS YOU CAN 4 00:04:21,040 --> 00:04:21,480 SEE HERE THERE'S OTHER 5 00:04:21,480 --> 00:04:27,360 INSTITUTIONS THAT FUND ALS. 6 00:04:27,360 --> 00:04:31,360 THERE'S $40 MILLION AS WELL 7 00:04:31,360 --> 00:04:34,360 THAT IS CONGRESSIONALLY 8 00:04:34,360 --> 00:04:39,120 MANDATED. NEXT SLIDE PLEASE. 9 00:04:39,120 --> 00:04:45,880 A NEW DEVELOPMENT WAS THE NIH 10 00:04:45,880 --> 00:04:53,520 COMMON FUND. AND THEN WE SET 11 00:04:53,520 --> 00:04:55,080 UP AN ACCELERATED SCIENCE 12 00:04:55,080 --> 00:04:57,560 PROGRAM WITH ALS. AND IT'S 13 00:04:57,560 --> 00:05:01,200 REALLY KIND OF OUT OF THE BOX 14 00:05:01,200 --> 00:05:07,960 PROJECTS AT VICKSBURG THERE'S 15 00:05:07,960 --> 00:05:15,200 TISSUE THAT I SHOULD SAY IS 16 00:05:15,200 --> 00:05:18,000 THAT HAVE ALLOWED THIS TYPE OF 17 00:05:18,000 --> 00:05:21,560 ANALYSIS TO BE DONE ON CELLS 18 00:05:21,560 --> 00:05:26,440 WHICH COULD REALLY MAKE THE 19 00:05:26,440 --> 00:05:31,080 PROBLEMS WORSE AND EXPAND THE 20 00:05:31,080 --> 00:05:32,840 MOTOR NEURONS. AND SEVER THE 21 00:05:32,840 --> 00:05:38,720 COURT WHICH ARE MORE EFFECTIVE 22 00:05:38,720 --> 00:05:40,840 IN THIS OFTEN OPENS UP THE 23 00:05:40,840 --> 00:05:45,200 POSSIBILITY OF HAVE TO 24 00:05:45,200 --> 00:05:47,360 INTERVENE IN THIS TYPE OF 25 00:05:47,360 --> 00:05:54,920 MANNER IN ALS IN THE FUTURE. 26 00:05:54,920 --> 00:05:55,400 THERE'S ALSO THE FUTURE 27 00:05:55,400 --> 00:06:02,320 LOOKING AT MACRO CROWDING AND 28 00:06:02,320 --> 00:06:04,120 ALS. THIS IS IDENTIFIED AS 29 00:06:04,120 --> 00:06:06,680 SOMETHING IS COMMON IN BOTH 30 00:06:06,680 --> 00:06:11,320 THE GENETIC FORM OF ALS. 31 00:06:11,320 --> 00:06:16,560 THOSE FINDINGS EXPAND TO THE 32 00:06:16,560 --> 00:06:18,760 NONGENETIC FORM ARE REALLY 33 00:06:18,760 --> 00:06:29,160 BEING HEAVILY THERE'S 34 00:06:41,560 --> 00:06:48,480 HOMEOSTASIS IN THE CELLS. AND 35 00:06:48,480 --> 00:06:59,000 THEY CAN TRY TO THIS PATHWAY. 36 00:07:06,840 --> 00:07:07,960 AND FINALLY UNIVERSITY OF 37 00:07:07,960 --> 00:07:10,880 MICHIGAN WAS DISCUSSING THIS 38 00:07:10,880 --> 00:07:11,400 LOOKING AT ENVIRONMENTAL 39 00:07:11,400 --> 00:07:12,320 EXPOSURES WITH ALS DISEASE 40 00:07:12,320 --> 00:07:13,440 RISK. AS WE MENTIONED THEY 41 00:07:13,440 --> 00:07:14,040 WERE LEARNING A LOT ABOUT THE 42 00:07:14,040 --> 00:07:18,000 GENETIC FORMS BUT WHAT IS 43 00:07:18,000 --> 00:07:19,120 DRIVING ALS FOR PEOPLE WHO DO 44 00:07:19,120 --> 00:07:23,080 NOT HAVE THIS HAS ALWAYS BEEN 45 00:07:23,080 --> 00:07:26,640 A MAJOR, MAJOR GAP IN 46 00:07:26,640 --> 00:07:29,000 KNOWLEDGE AND WE ARE HOPING TO 47 00:07:29,000 --> 00:07:34,640 EXPLORE THIS GAP. THERE'S 48 00:07:34,640 --> 00:07:36,040 DARK MATTER OF ENVIRONMENTAL 49 00:07:36,040 --> 00:07:36,640 EXPOSURE THAT LEADS TO THESE 50 00:07:36,640 --> 00:07:40,440 CONDITIONS. NEXT SLIDE 51 00:07:40,440 --> 00:07:41,680 PLEASE. WE HAVE THE CREATE 52 00:07:41,680 --> 00:07:46,800 SOURCE AND WHICH IS WHICH IS 53 00:07:46,800 --> 00:07:50,120 LOOKING AT ALS AND KIND OF A 54 00:07:50,120 --> 00:07:50,720 NATURAL HISTORY STUDY TRYING 55 00:07:50,720 --> 00:07:52,600 TO LOOK AT THE DIFFERENCE 56 00:07:52,600 --> 00:07:55,640 PHENOTYPES AND LOOKING AT 57 00:07:55,640 --> 00:07:56,920 BETTER CLINICAL OUTCOMES, 58 00:07:56,920 --> 00:07:57,600 SUBTYPING WITH DIFFERENT FORMS 59 00:07:57,600 --> 00:07:59,360 OF ALS AND LOOKING AT 60 00:07:59,360 --> 00:08:04,360 DEVELOPING BIOMARKERS. 61 00:08:04,360 --> 00:08:06,560 THERE'S NOW A FILAMENT LIGHT 62 00:08:06,560 --> 00:08:09,080 THAT SHOWS THE POTENTIAL 63 00:08:09,080 --> 00:08:14,640 BIOMARKER LINKED TO THE 64 00:08:14,640 --> 00:08:15,240 DISEASE. AND FOR THE VALUE OF 65 00:08:15,240 --> 00:08:22,720 THE THINGS PAN OUT THOSE 66 00:08:22,720 --> 00:08:24,360 THINGS THAT REDUCE THE 67 00:08:24,360 --> 00:08:24,920 FILAMENT LIGHT LEVELS ARE 68 00:08:24,920 --> 00:08:25,640 POTENTIALLY USEFUL IN LIMITING 69 00:08:25,640 --> 00:08:29,200 THE PROGRESSION OF THE 70 00:08:29,200 --> 00:08:30,080 DISEASE. THIS IS SOMETHING 71 00:08:30,080 --> 00:08:30,680 THAT HAS TO BE PROVEN FOR SURE 72 00:08:30,680 --> 00:08:35,960 BUT IT IS VERY HELPFUL 73 00:08:35,960 --> 00:08:39,200 DEVELOPMENT. OF COURSE THE 74 00:08:39,200 --> 00:08:41,200 CREATIVE NETWORK IS PART OF 75 00:08:41,200 --> 00:08:42,200 THE RARE DISEASE NETWORK HERE 76 00:08:42,200 --> 00:08:49,960 AT AND IH. NEXT SLIDE. THE 77 00:08:49,960 --> 00:08:53,440 CREATE CONSORTIUM WAS ALSO 78 00:08:53,440 --> 00:08:54,040 LOOKING AT DIVERSE POPULATION 79 00:08:54,040 --> 00:08:57,880 AND THEN GOING INTO SOUTH 80 00:08:57,880 --> 00:08:59,880 AFRICA. SOMETIMES CONDITIONS 81 00:08:59,880 --> 00:09:03,800 YOU CAN KIND OF HAVE AN 82 00:09:03,800 --> 00:09:07,800 EPIPHANY BY SETTING A 83 00:09:07,800 --> 00:09:09,360 PARTICULAR POPULATION WHERE IF 84 00:09:09,360 --> 00:09:11,000 YOU CAN IDENTIFY A PARTICULAR 85 00:09:11,000 --> 00:09:11,680 CORE OF ALS AND AN 86 00:09:11,680 --> 00:09:12,200 ENVIRONMENTAL CORE FOR 87 00:09:12,200 --> 00:09:15,320 INSTANCE IN SOUTH AFRICA. 88 00:09:15,320 --> 00:09:15,880 THIS COULD THEN BE TESTED IN 89 00:09:15,880 --> 00:09:22,960 THE UNITED STATES. OR 90 00:09:22,960 --> 00:09:23,440 ACTUALLY NEW GENETICS 91 00:09:23,440 --> 00:09:24,680 UNDERPINNING FROM ALS AS IT IS 92 00:09:24,680 --> 00:09:25,880 WITH THE DIVERSE POPULATION 93 00:09:25,880 --> 00:09:26,560 LIKE THIS WILL BE POTENTIALLY 94 00:09:26,560 --> 00:09:33,760 HELPFUL. NEXT SLIDE. GETTING 95 00:09:33,760 --> 00:09:35,480 BACK TO WHAT WE HAD BEEN 96 00:09:35,480 --> 00:09:37,960 SAYING THE LAST COUPLE OF 97 00:09:37,960 --> 00:09:41,040 MINUTES IS THAT TEN-15% OF ALS 98 00:09:41,040 --> 00:09:42,800 IS FAMILIAL AND THERE'S 13 OR 99 00:09:42,800 --> 00:09:45,800 EXCUSE ME 30 MUTANT GENES HAVE 100 00:09:45,800 --> 00:09:47,600 IDENTIFIED SO FAR. THE 101 00:09:47,600 --> 00:09:48,400 FINDING OF A GENETIC MUTATION 102 00:09:48,400 --> 00:09:50,200 REALLY OPENS UP THE DOOR TO 103 00:09:50,200 --> 00:09:51,200 GET INTO THE DETAILS OF THE 104 00:09:51,200 --> 00:09:57,240 BIOLOGY OF THE DISEASE. THERE 105 00:09:57,240 --> 00:09:57,800 YOU CAN STUDY FOR THE 106 00:09:57,800 --> 00:09:59,640 BEGINNING OF THE STAGES TO SEE 107 00:09:59,640 --> 00:10:02,480 WHAT THAT DOES TO MOTOR NEURON 108 00:10:02,480 --> 00:10:04,200 CELLS AND THERE YOU CAN LOOK 109 00:10:04,200 --> 00:10:05,720 AT CELLS THAT HAVE BEEN TURNED 110 00:10:05,720 --> 00:10:07,200 INTO MOTOR NEURONS FROM SKIN 111 00:10:07,200 --> 00:10:08,520 CELLS TAKEN FROM PEOPLE AND 112 00:10:08,520 --> 00:10:09,600 THE GENETICS REALLY OPENED THE 113 00:10:09,600 --> 00:10:15,640 DOOR TO USE THE TOOLS OF 114 00:10:15,640 --> 00:10:16,240 SCIENCE TO UNDERSTAND THE 115 00:10:16,240 --> 00:10:16,760 DISEASE. IT ALSO LEADS TO 116 00:10:16,760 --> 00:10:19,400 THERAPIES AND THAT IS WHERE 117 00:10:19,400 --> 00:10:21,840 SOME OF THE BIGGEST 118 00:10:21,840 --> 00:10:23,040 BREAKTHROUGHS MAKE FOR 119 00:10:23,040 --> 00:10:23,680 EFFECTIVE THERAPIES AND MAY BE 120 00:10:23,680 --> 00:10:30,400 COMING IN THE NEAR FUTURE. 121 00:10:30,400 --> 00:10:33,040 BUT AGAIN, THE MAJORITY 122 00:10:33,040 --> 00:10:33,800 TEN-20% ARE FAMILIAL WHICH 123 00:10:33,800 --> 00:10:36,000 LEADS 85% WHICH ARE NOT. AND 124 00:10:36,000 --> 00:10:38,800 THAT WOULD CAUSE A SPORADIC, 125 00:10:38,800 --> 00:10:40,720 NON- FAMILIAR, SO TRYING TO 126 00:10:40,720 --> 00:10:44,600 UNDERSTAND WHAT ARE THE 127 00:10:44,600 --> 00:10:45,200 NONGENETIC FACTORS THAT CAUSE 128 00:10:45,200 --> 00:10:47,160 ALS AND CONTINUE ITS 129 00:10:47,160 --> 00:10:49,320 PROGRESSION, AS I MENTIONED, 130 00:10:49,320 --> 00:10:53,320 THE REAL CHALLENGE LOOKING 131 00:10:53,320 --> 00:10:54,960 FORWARD. THIS IS SIMPLY NO 132 00:10:54,960 --> 00:10:56,640 PROBLEM WITH ALS IS A PROBLEM 133 00:10:56,640 --> 00:10:58,880 WITH MOST NEUROLOGICAL 134 00:10:58,880 --> 00:10:59,440 DISEASES. WE HAVE RECENTLY 135 00:10:59,440 --> 00:11:03,120 STARTED A NEW OFFICE FOR THIS 136 00:11:03,120 --> 00:11:07,360 TOXICOLOGY STUDY AND THE GOAL 137 00:11:07,360 --> 00:11:09,280 IS TO ADVANCE THE 138 00:11:09,280 --> 00:11:09,840 UNDERSTANDING OF THE NON- 139 00:11:09,840 --> 00:11:10,480 INHERITED FACTORS COLLECTIVELY 140 00:11:10,480 --> 00:11:16,000 CALLED THE NEURAL DISPOSABLE. 141 00:11:16,000 --> 00:11:16,560 THAT COULD BE FACTORS IN THE 142 00:11:16,560 --> 00:11:22,560 ENVIRONMENT LIKE TOXICS AND AN 143 00:11:22,560 --> 00:11:23,480 INTERPLAY BETWEEN SOMETHING IN 144 00:11:23,480 --> 00:11:24,280 THE ENVIRONMENT IN A 145 00:11:24,280 --> 00:11:28,160 PARTICULAR GENE THAT PEOPLE 146 00:11:28,160 --> 00:11:28,680 HAVE. OR THERE COULD BE 147 00:11:28,680 --> 00:11:29,560 BEHAVIORAL FACTORS AS WELL 148 00:11:29,560 --> 00:11:34,360 THAT CONTRIBUTE TO THIS MIX OF 149 00:11:34,360 --> 00:11:38,280 GENE ENVIRONMENT INTERACTIONS. 150 00:11:38,280 --> 00:11:44,040 NEXT SLIDE PLEASE. THERE ARE 151 00:11:44,040 --> 00:11:44,640 A NUMBER OF GENE -BASED 152 00:11:44,640 --> 00:11:46,520 THERAPIES COMING, WHEN 153 00:11:46,520 --> 00:11:52,280 MENTIONED. THE GENETIC WAYS 154 00:11:52,280 --> 00:11:52,880 TO WHICH OF THESE THAT CAUSE 155 00:11:52,880 --> 00:11:59,160 TROUBLE CAN BE BLOCKED. THERE 156 00:11:59,160 --> 00:12:03,400 IS THE ANSWER I SENT NUCLEAR 157 00:12:03,400 --> 00:12:07,440 TIDE ARE SUPER OXIDE THIS ALS. 158 00:12:07,440 --> 00:12:11,720 THERE'S ANTIRACIST ANTISENSE 159 00:12:11,720 --> 00:12:15,360 NUCLEAR TIDE FOR THE SC NINE 160 00:12:15,360 --> 00:12:16,600 OH RF 72 LATE, WHICH IS THE 161 00:12:16,600 --> 00:12:22,760 MOST COMMON GENETIC FORM. AND 162 00:12:22,760 --> 00:12:24,400 THEN THERE IS THE ANTI- CENSUS 163 00:12:24,400 --> 00:12:25,000 WELL FOR THE OTHER LINK OF ALS 164 00:12:25,000 --> 00:12:29,240 WHICH IS LESS COMMON GENETIC 165 00:12:29,240 --> 00:12:35,800 FORM AND ANOTHER MICRO RNA FOR 166 00:12:35,800 --> 00:12:37,600 THE SOD ONE LINKED ALS FROM 167 00:12:37,600 --> 00:12:39,640 THIS COMPANY. SO THE AAS 168 00:12:39,640 --> 00:12:42,000 THOSE ARE WALKING OR THE IDEA 169 00:12:42,000 --> 00:12:45,880 IS TO BLOCK THE BAD EFFECTS OF 170 00:12:45,880 --> 00:12:48,320 THESE MUTATED GENES. AS YOU 171 00:12:48,320 --> 00:12:53,920 CAN SEE HERE ONE OF THE 172 00:12:53,920 --> 00:12:55,360 BIGGEST PUSHES IN THE ALS 173 00:12:55,360 --> 00:12:57,920 TREATMENT IS THE HOPE OF THESE 174 00:12:57,920 --> 00:12:59,480 WOULD HAVE HIGH EFFECT SIZES. 175 00:12:59,480 --> 00:13:00,160 ALTHOUGH THERE ARE STILL LOTS 176 00:13:00,160 --> 00:13:01,840 OF QUESTIONS ABOUT HOW THESE 177 00:13:01,840 --> 00:13:04,360 WORK AND HOW YOU GET INTO THE 178 00:13:04,360 --> 00:13:06,200 CELLS THAT YOU NEED TO GET 179 00:13:06,200 --> 00:13:08,200 THEM INTO AND HOW OFTEN DO YOU 180 00:13:08,200 --> 00:13:10,200 HAVE TO TREAT? OR THE 181 00:13:10,200 --> 00:13:11,120 POTENTIAL TOXICITIES OF THESE 182 00:13:11,120 --> 00:13:11,640 TYPES OF THERAPIES. IN 183 00:13:11,640 --> 00:13:15,040 PARTICULAR, THE NERVOUS 184 00:13:15,040 --> 00:13:15,640 SYSTEM. THERE'S A LOT TO 185 00:13:15,640 --> 00:13:19,120 LEARN BUT THIS IS THE KIND OF 186 00:13:19,120 --> 00:13:21,040 THING THAT WE FIGURE OUT THE 187 00:13:21,040 --> 00:13:23,120 ISSUES IT SHOULD REALLY WORK 188 00:13:23,120 --> 00:13:24,680 FOR THESE CONDITIONS. OR AT 189 00:13:24,680 --> 00:13:25,240 LEAST THAT'S WHAT WE THINK. 190 00:13:25,240 --> 00:13:32,400 NEXT SLIDE. WE HAVE FOR THE 191 00:13:32,400 --> 00:13:35,000 FORMS OF ALS AND OTHER REALLY 192 00:13:35,000 --> 00:13:36,720 WHERE GENETIC DISORDERS, AS I 193 00:13:36,720 --> 00:13:37,320 MENTIONED, THERE'S 30 GENES 194 00:13:37,320 --> 00:13:43,120 FOR ALS. WE TALKED ABOUT ASO 195 00:13:43,120 --> 00:13:44,000 TREATMENTS FOR THREE OF THOSE 196 00:13:44,000 --> 00:13:46,600 30. THE OTHERS, MANY OF WHICH 197 00:13:46,600 --> 00:13:48,680 ARE EXTREMELY RARE. SO, WE DO 198 00:13:48,680 --> 00:13:49,240 NOT CURRENTLY HAVE COMPANIES 199 00:13:49,240 --> 00:13:52,880 GOING AFTER THE TREATMENTS. 200 00:13:52,880 --> 00:13:53,440 WE HAD THIS NETWORK WHICH IS 201 00:13:53,440 --> 00:13:57,720 BEING SET UP NOW FOR WHAT WE 202 00:13:57,720 --> 00:13:59,040 CALL ULTRA RARE GENETIC 203 00:13:59,040 --> 00:14:00,080 DISORDERS, SUCH AS SOME OF 204 00:14:00,080 --> 00:14:04,960 THESE RARE FORMS OF ALS. THEY 205 00:14:04,960 --> 00:14:06,880 DEVELOP NOT JUST ASO BUT GENE 206 00:14:06,880 --> 00:14:08,000 DEVELOPING TREATMENTS FOR 207 00:14:08,000 --> 00:14:09,080 THESE TYPES OF DISORDERS. 208 00:14:09,080 --> 00:14:10,280 THIS IS NOW OPEN AND WERE 209 00:14:10,280 --> 00:14:13,280 HOPING TO GET APPLICATIONS TO 210 00:14:13,280 --> 00:14:16,440 TRY SOME OF THESE THERE 211 00:14:16,440 --> 00:14:17,960 APPEARS WITH SOME OF THE ULTRA 212 00:14:17,960 --> 00:14:22,040 RARE FORMS OF ALS. NEXT 213 00:14:22,040 --> 00:14:25,840 SLIDE. THIS ONE IS AGAIN 214 00:14:25,840 --> 00:14:26,400 SOMETHING THAT WE THINK IS 215 00:14:26,400 --> 00:14:31,000 IMPORTANT TO HELP PEOPLE WITH 216 00:14:31,000 --> 00:14:33,240 ALS. IF WE REALLY CANNOT 217 00:14:33,240 --> 00:14:37,240 PREVENT THE PROGRESSION AND 218 00:14:37,240 --> 00:14:39,200 ONE OF THE BIGGEST PROBLEMS IS 219 00:14:39,200 --> 00:14:40,560 COMMUNICATIONS AS TIME GOES 220 00:14:40,560 --> 00:14:41,960 ON. SO, WHEN THE BRAIN 221 00:14:41,960 --> 00:14:42,480 INITIATIVE AND STUDIES ARE 222 00:14:42,480 --> 00:14:46,160 GOING ON WHERE GRIDS OF 223 00:14:46,160 --> 00:14:50,200 ELECTRODES ARE PLACED OVER THE 224 00:14:50,200 --> 00:14:51,200 PART OF THE BRAIN THAT IS PART 225 00:14:51,200 --> 00:14:57,040 OF PRODUCING SPEECH. IT TURNS 226 00:14:57,040 --> 00:14:59,280 OUT IF ONE IS PARALYZED AND 227 00:14:59,280 --> 00:15:00,680 CANNOT MOVE THE MUSCLES ONE 228 00:15:00,680 --> 00:15:01,280 CAN STILL TRY TO SPEAK AND 229 00:15:01,280 --> 00:15:05,200 ACTIVATE THIS PART OF THE 230 00:15:05,200 --> 00:15:08,520 BRAIN. IN THIS STUDY THEY 231 00:15:08,520 --> 00:15:08,920 WERE ABLE TO USE THE 232 00:15:08,920 --> 00:15:09,880 ELECTRICAL ACTIVITY THAT COMES 233 00:15:09,880 --> 00:15:13,800 OUT OF THE BRAIN WHEN SOMEONE 234 00:15:13,800 --> 00:15:16,960 SPEAKS TO CREATE LANGUAGE ON A 235 00:15:16,960 --> 00:15:19,160 COMPUTER. SO, IT IS BASICALLY 236 00:15:19,160 --> 00:15:22,600 BYPASSING THE NERVES FROM THE 237 00:15:22,600 --> 00:15:23,120 BRAINS TO THE MUSCLES AND 238 00:15:23,120 --> 00:15:23,720 GOING STRAIGHT FROM THE BRAIN 239 00:15:23,720 --> 00:15:29,480 TO THE COMPUTER TO GENERATE 240 00:15:29,480 --> 00:15:33,280 SPEECH WE WOULD REALLY LIKE TO 241 00:15:33,280 --> 00:15:34,040 STOP THE DISEASE FROM EVER 242 00:15:34,040 --> 00:15:35,080 GETTING TO THIS PLACE BUT I 243 00:15:35,080 --> 00:15:37,240 THINK THAT WE ALWAYS HAVE TO 244 00:15:37,240 --> 00:15:43,600 THINK ABOUT INVESTING AND NOT 245 00:15:43,600 --> 00:15:44,640 JUST THE FUTURE TREATMENTS BUT 246 00:15:44,640 --> 00:15:45,880 ALSO HOW TO BEST HELP PEOPLE 247 00:15:45,880 --> 00:15:48,480 WHO HAVE THIS DISEASE NOW 248 00:15:48,480 --> 00:15:49,120 BEFORE THESE TREATMENTS BECOME 249 00:15:49,120 --> 00:15:59,640 EFFECTIVE. NEXT SLIDE PLEASE 250 00:16:00,040 --> 00:16:02,040 AS YOU PROBABLY KNOW, 251 00:16:02,040 --> 00:16:03,600 PRESIDENT BIDEN ON 252 00:16:03,600 --> 00:16:04,240 DECEMBER 201st, SIGNED THE 253 00:16:04,240 --> 00:16:04,840 ACCELERATED AXIS FOR CRITICAL 254 00:16:04,840 --> 00:16:09,560 THERAPIES ACT. AND IH IS 255 00:16:09,560 --> 00:16:11,280 GEARED UP TO FIND AND EXPAND 256 00:16:11,280 --> 00:16:17,680 ACCESS AND STUDY WITH A DRUG 257 00:16:17,680 --> 00:16:18,320 THAT IS CURRENTLY IN TRIALS ON 258 00:16:18,320 --> 00:16:23,240 THE HEEL OF THE ALS PLATFORM. 259 00:16:23,240 --> 00:16:27,440 THIS WILL ALLOW FOR PEOPLE WHO 260 00:16:27,440 --> 00:16:29,200 DO NOT QUALIFY FOR THE 261 00:16:29,200 --> 00:16:30,800 CLINICAL TRIALS OR OUT OF THE 262 00:16:30,800 --> 00:16:31,600 CLINICAL TRIALS TO BE TREATED 263 00:16:31,600 --> 00:16:37,760 WITH THIS DRUG. ACTIVE ALS 264 00:16:37,760 --> 00:16:39,000 ALSO CALLED FOR A 265 00:16:39,000 --> 00:16:39,520 PUBLIC-PRIVATE PARTNERSHIP 266 00:16:39,520 --> 00:16:40,080 BETWEEN THE FDA AND THE NIH 267 00:16:40,080 --> 00:16:43,760 AND WE HAVE BEEN WORKING THERE 268 00:16:43,760 --> 00:16:44,320 AND WERE VERY EXCITED ABOUT 269 00:16:44,320 --> 00:16:49,200 THIS NEW PARTNERSHIP. IT WILL 270 00:16:49,200 --> 00:16:51,360 BE ORCHESTRATED THROUGH THE 271 00:16:51,360 --> 00:16:55,200 CLINICAL PATH FOR RARE 272 00:16:55,200 --> 00:16:57,640 NEURODEGENERATIVE DISEASES AND 273 00:16:57,640 --> 00:16:59,040 THIS IS THE CRITICAL PATH 274 00:16:59,040 --> 00:17:00,360 INSTITUTE THAT THE FDA HAS 275 00:17:00,360 --> 00:17:03,120 BEEN WORKING FOR A NUMBER OF 276 00:17:03,120 --> 00:17:06,960 DECADES NOW. THEY CAN BE ABLE 277 00:17:06,960 --> 00:17:08,480 TO BRING THE FDA AND NIH 278 00:17:08,480 --> 00:17:08,960 PATIENT COMMITTEES AND 279 00:17:08,960 --> 00:17:11,120 ADVOCACY OVER THIS 280 00:17:11,120 --> 00:17:11,920 PHARMACEUTICAL COMPANY IN THE 281 00:17:11,920 --> 00:17:16,720 BIO TEXT ALTOGETHER WORKED OUT 282 00:17:16,720 --> 00:17:20,640 HOW TO MAKE THE PROCESS OF 283 00:17:20,640 --> 00:17:21,120 THERAPY DEVELOPMENT AND 284 00:17:21,120 --> 00:17:23,120 APPROVAL OF EFFECTIVE 285 00:17:23,120 --> 00:17:27,360 THERAPIES MORE STREAMLINED. 286 00:17:27,360 --> 00:17:28,160 THIS CAN BRING ALL THE DATA 287 00:17:28,160 --> 00:17:29,840 TOGETHER FROM ALL OF THE 288 00:17:29,840 --> 00:17:31,960 DIFFERENT SOURCES FROM WHERE 289 00:17:31,960 --> 00:17:35,960 WE START AND THEN ANALYZE THAT 290 00:17:35,960 --> 00:17:38,680 DATA AND THEN WE CAN TO MOVE 291 00:17:38,680 --> 00:17:39,240 THINGS FORWARD IN THE FUTURE 292 00:17:39,240 --> 00:17:41,080 BASED ON WHAT HAS BEEN DONE IN 293 00:17:41,080 --> 00:17:42,120 THE PAST. I THINK THAT MIGHT 294 00:17:42,120 --> 00:17:47,640 BE ONE OF OUR FIRST ATTEMPTS 295 00:17:47,640 --> 00:17:53,240 HERE. IT IS ALWAYS FOCUSED ON 296 00:17:53,240 --> 00:17:54,680 PATIENT AND DRUG DEVELOPMENT 297 00:17:54,680 --> 00:17:55,920 TO HELP PATIENTS. THE FDA 298 00:17:55,920 --> 00:17:56,480 ALSO SUPPORTS WORKING WITH 299 00:17:56,480 --> 00:17:59,600 THEM ON THE RARE GENETIC 300 00:17:59,600 --> 00:18:04,560 DISEASE PROGRAM. THEY HAVE 301 00:18:04,560 --> 00:18:05,240 FUNDED AND JUST LAUNCHED A 302 00:18:05,240 --> 00:18:09,480 NATURAL HISTORY STUDY IN ALS. 303 00:18:09,480 --> 00:18:11,640 THEY ALSO ARE STUDYING OTHER 304 00:18:11,640 --> 00:18:15,560 TOOLS IN ALS WITH THIS 305 00:18:15,560 --> 00:18:18,280 PARTNERSHIP. COMING UP AFTER 306 00:18:18,280 --> 00:18:22,240 THE STRATEGIC PLAN THERE WILL 307 00:18:22,240 --> 00:18:24,400 BE A NATIONAL ACADEMY OF 308 00:18:24,400 --> 00:18:26,240 SCIENCES STUDY AND RESEARCH 309 00:18:26,240 --> 00:18:27,600 CARE AND SERVICES FOR ALS. 310 00:18:27,600 --> 00:18:28,200 THIS WAS COMMISSIONED BY NIH. 311 00:18:28,200 --> 00:18:31,640 THIS GROUP WILL THEN 312 00:18:31,640 --> 00:18:33,680 IDENTIFYING KEY ACTIONS FOR 313 00:18:33,680 --> 00:18:37,880 THE PUBLIC, PRIVATE, AND 314 00:18:37,880 --> 00:18:38,480 NONPUBLIC SECTORS. THIS WILL 315 00:18:38,480 --> 00:18:39,840 BUILD UPON THE RESEARCH 316 00:18:39,840 --> 00:18:42,080 PRIORITIES YOU HEAR ABOUT 317 00:18:42,080 --> 00:18:42,840 TODAY. AND THAT INCLUDE 318 00:18:42,840 --> 00:18:43,400 RECOMMENDATIONS FOR CARE AND 319 00:18:43,400 --> 00:18:45,440 SERVICES AS WELL AS THE PLAN 320 00:18:45,440 --> 00:18:51,880 THAT YOU HEAR TODAY WHICH IS 321 00:18:51,880 --> 00:18:52,480 FOCUSED ENTIRELY ON RESEARCH 322 00:18:52,480 --> 00:18:55,360 DEVELOPMENT THERAPY PROGRAMS. 323 00:18:55,360 --> 00:18:56,200 THEY WILL HAVE A COMMITTEE, 324 00:18:56,200 --> 00:19:00,400 SOMETIMES UP TO 15 EXPERTS. 325 00:19:00,400 --> 00:19:03,760 AND THEN THE SCIENCE OF ALS 326 00:19:03,760 --> 00:19:04,800 AND DEVELOPMENT REGULATION BUT 327 00:19:04,800 --> 00:19:05,360 WILL ALSO INCLUDE PEOPLE WHO 328 00:19:05,360 --> 00:19:10,080 HAVE LIVED WITH ALS, PEOPLE 329 00:19:10,080 --> 00:19:11,800 WHO CARE FOR PEOPLE WITH ALS 330 00:19:11,800 --> 00:19:12,400 AND PEOPLE WITH GENETIC RISKS 331 00:19:12,400 --> 00:19:21,000 WITH THE DISEASE. IT WILL 332 00:19:21,000 --> 00:19:22,120 WORK ON DELIVERY SUPPORT AND 333 00:19:22,120 --> 00:19:22,640 ACCESS AND ARE LOOKING FOR 334 00:19:22,640 --> 00:19:27,200 NOMINATION OF PEOPLE FOR THIS 335 00:19:27,200 --> 00:19:31,280 GROUP AN ILLNESS OR IF YOU 336 00:19:31,280 --> 00:19:32,760 KNOW SOMEBODY WHO'D BE 337 00:19:32,760 --> 00:19:34,320 INTERESTED, PLEASE SUBMIT 338 00:19:34,320 --> 00:19:35,360 NOMINATIONS BY NOVEMBER 4th 339 00:19:35,360 --> 00:19:36,320 ON THIS WEBSITE LISTED. WE 340 00:19:36,320 --> 00:19:37,720 WILL TRY TO PUT THIS IN THE 341 00:19:37,720 --> 00:19:39,280 CHAT SO IT WILL BE AVAILABLE 342 00:19:39,280 --> 00:19:39,880 THROUGHOUT THE MEETING TODAY. 343 00:19:39,880 --> 00:19:46,960 NEXT SLIDE. SO, NOW WERE 344 00:19:46,960 --> 00:19:47,440 GOING TO HEAR FROM THE 345 00:19:47,440 --> 00:19:52,240 STRATEGIC PLAN AND THIS BEGAN 346 00:19:52,240 --> 00:19:54,080 AS A PUBLIC REQUEST 347 00:19:54,080 --> 00:19:56,840 INFORMATION AND RECEIVED OVER 348 00:19:56,840 --> 00:20:00,480 100 RESPONSES AND LOOKED AT 349 00:20:00,480 --> 00:20:01,000 THE FORMS AND THE DRAFT 350 00:20:01,000 --> 00:20:06,160 PRIORITIES HAVE BEEN MET ON 351 00:20:06,160 --> 00:20:06,640 SEVERAL OCCASIONS AND 352 00:20:06,640 --> 00:20:07,240 INDIVIDUALLY AND ONE GROUP AT 353 00:20:07,240 --> 00:20:08,960 A TIME. AND THEY ALSO MET 354 00:20:08,960 --> 00:20:09,520 WITH ALL OF THE DIFFERENT 355 00:20:09,520 --> 00:20:11,240 GROUPS AND TRADED THEIR 356 00:20:11,240 --> 00:20:15,280 THOUGHTS BACK AND FORTH. WERE 357 00:20:15,280 --> 00:20:16,440 GOING TO HEAR IF THE SCIENCE 358 00:20:16,440 --> 00:20:19,120 PEOPLE IN THE TRANSLATIONAL 359 00:20:19,120 --> 00:20:23,960 SCIENCE AND MOVING THE 360 00:20:23,960 --> 00:20:25,280 DISCOVERIES TO THERAPIES IN 361 00:20:25,280 --> 00:20:26,960 THE CLINICAL GROUP AND THE 362 00:20:26,960 --> 00:20:27,680 QUALITY OF LIFE GROUP AND THEN 363 00:20:27,680 --> 00:20:32,440 A SPECIAL GROUP FOCUSING ON 364 00:20:32,440 --> 00:20:33,040 HOW TO BE FORGED MORE 365 00:20:33,040 --> 00:20:33,600 EFFECTIVE PARTNERSHIPS AND 366 00:20:33,600 --> 00:20:34,200 COLLABORATIONS IN THE FUTURE? 367 00:20:34,200 --> 00:20:37,720 WE ASKED FOR THEM WE ASKED 368 00:20:37,720 --> 00:20:42,040 THEM TO BE CRITICAL AND MAKE 369 00:20:42,040 --> 00:20:42,760 COMPREHENSIVE LIFTS FOR 370 00:20:42,760 --> 00:20:43,360 STRAIGHT TO THE TOP THEIR TOP 371 00:20:43,360 --> 00:20:49,800 THREE PRIORITIES IT'S REALLY 372 00:20:49,800 --> 00:20:50,360 IMPORTANT CERTAINLY FOR ANY 373 00:20:50,360 --> 00:20:53,560 AGENCY SUCH AS US TO HAVE A 374 00:20:53,560 --> 00:20:58,840 SENSE OF WHAT RISES TO THE 375 00:20:58,840 --> 00:21:00,280 TOP? THOSE ARE KIND OF THE 376 00:21:00,280 --> 00:21:02,840 THINGS THAT YOU WOULD GO AFTER 377 00:21:02,840 --> 00:21:03,840 FIRST. NOW HERE AT THE PUBLIC 378 00:21:03,840 --> 00:21:05,520 WORKSHOP AND THEN AFTER THIS 379 00:21:05,520 --> 00:21:06,440 THERE WILL BE A PUBLIC 380 00:21:06,440 --> 00:21:07,200 COMMENT. THAT WILL BE OPEN 381 00:21:07,200 --> 00:21:13,480 FOR ABOUT ONE MONTH. SO, THE 382 00:21:13,480 --> 00:21:14,920 COMMENTS THAT COME IN WILL BE 383 00:21:14,920 --> 00:21:15,920 USED BY THE GROUP TO REVISE 384 00:21:15,920 --> 00:21:19,120 PRIORITIES AND THEN BRING A 385 00:21:19,120 --> 00:21:22,600 FINAL VERSION TO THE ADVISORY 386 00:21:22,600 --> 00:21:24,440 COUNCIL AND THE FIRST WEEK OF 387 00:21:24,440 --> 00:21:30,680 FEBRUARY 2023. NEXT SLIDE. I 388 00:21:30,680 --> 00:21:32,440 WOULD LIKE TO THINK ANYONE WHO 389 00:21:32,440 --> 00:21:33,720 HAS WORKED ON THIS. AS I 390 00:21:33,720 --> 00:21:34,240 MENTIONED THERE'S A LOT OF 391 00:21:34,240 --> 00:21:37,000 PEOPLE ON THIS LIST AND SOME 392 00:21:37,000 --> 00:21:42,600 PEOPLE HAVE ALS AND SOME 393 00:21:42,600 --> 00:21:43,200 PEOPLE CARE WITH PEOPLE WITH 394 00:21:43,200 --> 00:21:46,840 ALS AND SOME PEOPLE HAVE SPENT 395 00:21:46,840 --> 00:21:47,560 DECADES TRYING TO DEVELOP A 396 00:21:47,560 --> 00:21:51,600 BETTER TREATMENT FOR THIS 397 00:21:51,600 --> 00:21:54,160 DISEASE. WE'VE BEEN INVOLVED 398 00:21:54,160 --> 00:21:59,000 IN MANY DIFFERENT DISEASE 399 00:21:59,000 --> 00:21:59,720 RELATED COMMUNITIES. THE ALS 400 00:21:59,720 --> 00:22:00,320 DISEASE RESEARCH COMMITTEE IS 401 00:22:00,320 --> 00:22:05,440 REALLY A SPECIAL GROUP AND 402 00:22:05,440 --> 00:22:05,920 THEN DEDICATION WHICH IS 403 00:22:05,920 --> 00:22:06,480 MOTIVATED BY THE URGENCY OF 404 00:22:06,480 --> 00:22:08,120 THE DISEASE AND THE PEOPLE 405 00:22:08,120 --> 00:22:11,040 THEY KNOW THAT HAVE THE 406 00:22:11,040 --> 00:22:13,600 DISEASE AND TRY TO TAKE CARE 407 00:22:13,600 --> 00:22:14,680 OF. IT'S REALLY UNMATCHED. 408 00:22:14,680 --> 00:22:16,680 SO THANK YOU VERY MUCH FOR 409 00:22:16,680 --> 00:22:19,440 EVERYONE'S HELP FROM THIS 410 00:22:19,440 --> 00:22:21,880 EXPERIENCE TO GROUP IN PUTTING 411 00:22:21,880 --> 00:22:29,080 TOGETHER THIS STRATEGIC PLAN. 412 00:22:29,080 --> 00:22:30,160 NEXT SLIDE. AS THE PLAN COMES 413 00:22:30,160 --> 00:22:31,120 IN THE NIH WILL REVIEW THE 414 00:22:31,120 --> 00:22:34,400 PORTFOLIO THAT WE HAVE LOOKING 415 00:22:34,400 --> 00:22:35,080 FOR PRIORITIES AND THAT WE 416 00:22:35,080 --> 00:22:35,640 HAVE A NUMBER OF LEVERS THAT 417 00:22:35,640 --> 00:22:40,360 WE CAN PULL. WE CAN PUSH 418 00:22:40,360 --> 00:22:41,040 PEOPLE TO LOOK AT TOPICS THAT 419 00:22:41,040 --> 00:22:47,840 HAVE COME OUT OF THIS. I 420 00:22:47,840 --> 00:22:50,880 SUSPECT THERE WILL BE A LOT OF 421 00:22:50,880 --> 00:22:54,080 INTEREST IN MOVING THE SEARCH 422 00:22:54,080 --> 00:22:56,080 FROM THE COMMUNITY AND THESE 423 00:22:56,080 --> 00:22:58,080 TOPICS. WE HAVE THE ABILITY 424 00:22:58,080 --> 00:22:58,800 TO USE SPECIAL PROGRAM 425 00:22:58,800 --> 00:22:59,400 PRIORITY FUNDING TO MOVE SOME 426 00:22:59,400 --> 00:23:04,520 OF THESE THINGS FORWARD. WE 427 00:23:04,520 --> 00:23:05,840 CAN MODIFY CURRENT PROGRAMS 428 00:23:05,840 --> 00:23:06,360 AND DEVELOP NEW FUNDING 429 00:23:06,360 --> 00:23:10,080 OPPORTUNITIES AND WE CAN HOLD 430 00:23:10,080 --> 00:23:11,760 WORKSHOPS AND WORK WITH THE 431 00:23:11,760 --> 00:23:12,920 PUBLIC BY PARTNERSHIP TO MOVE 432 00:23:12,920 --> 00:23:15,880 THINGS FORWARD. WE WANT TO 433 00:23:15,880 --> 00:23:17,440 START BY MENTIONING THAT 434 00:23:17,440 --> 00:23:18,480 PEOPLE WHO HAVE GOOD IDEAS FOR 435 00:23:18,480 --> 00:23:21,280 ALS RESEARCH YOU DON'T HAVE TO 436 00:23:21,280 --> 00:23:27,400 WAIT. AND I DS IS OPEN TO 437 00:23:27,400 --> 00:23:28,400 APPLICATIONS INTO THREE TIMES 438 00:23:28,400 --> 00:23:30,080 A YEAR THEY COME IN SO IF 439 00:23:30,080 --> 00:23:30,920 YOU'RE INTERESTED CONTACT THE 440 00:23:30,920 --> 00:23:33,840 PROGRAM OFFICIALS TO HELP YOU 441 00:23:33,840 --> 00:23:36,560 FIND THE RIGHT PROGRAM THROUGH 442 00:23:36,560 --> 00:23:37,240 THE GRANT APPLICATION. WITH 443 00:23:37,240 --> 00:23:38,520 THAT, I WOULD LIKE TO AGAIN 444 00:23:38,520 --> 00:23:42,040 SAY THANKS EVERYBODY FOR 445 00:23:42,040 --> 00:23:43,080 LISTENING. THANK YOU TO THE 446 00:23:43,080 --> 00:23:43,720 FOLKS WHO WORK SO HARD ON THIS 447 00:23:43,720 --> 00:23:47,640 AND TURN IT BACK TO ROBERT 448 00:23:47,640 --> 00:23:48,160 BROWN'S AUNT RITA SET THE 449 00:23:48,160 --> 00:23:53,880 CHAIRS OF THE ORTHOPEDIC 450 00:23:53,880 --> 00:23:55,880 GROUP. 451 00:23:55,880 --> 00:23:57,600 >>: THANK YOU VERY MUCH 452 00:23:57,600 --> 00:23:59,320 WALTER I'M A NEUROLOGIST AT 453 00:23:59,320 --> 00:24:00,680 THE UNIVERSITY OF 454 00:24:00,680 --> 00:24:01,080 MASSACHUSETTS WITH 455 00:24:01,080 --> 00:24:01,560 LONG-STANDING EXPERIENCE 456 00:24:01,560 --> 00:24:02,160 THINKING ABOUT ASPECTS OF ALS, 457 00:24:02,160 --> 00:24:08,520 CAUSE, AND THERAPY. I MUST 458 00:24:08,520 --> 00:24:10,280 SAY IT'S A GREAT PRIVILEGE TO 459 00:24:10,280 --> 00:24:11,880 BE IN ALS PHYSICIAN AND IT'S A 460 00:24:11,880 --> 00:24:13,280 GREAT PRIVILEGE TO BE PART OF 461 00:24:13,280 --> 00:24:14,920 THIS CAUSE TODAY. IT IS 462 00:24:14,920 --> 00:24:16,480 INCREDIBLY EXCITING TIME FOR 463 00:24:16,480 --> 00:24:18,520 THOSE OF US IN ALS RESEARCH. 464 00:24:18,520 --> 00:24:20,640 THERE ARE MORE TO USE WALTER'S 465 00:24:20,640 --> 00:24:21,240 TERM, APART FROM THE TOOLSET 466 00:24:21,240 --> 00:24:25,120 IN SCIENCE ON THE TABLE NO USE 467 00:24:25,120 --> 00:24:25,600 FOR THINKING AND IT'S 468 00:24:25,600 --> 00:24:27,880 INCREDIBLY EXCITING TO BE PART 469 00:24:27,880 --> 00:24:31,120 OF THIS PROCESS WITH THE NIH 470 00:24:31,120 --> 00:24:32,440 TRYING TO ESTABLISH THE MOST 471 00:24:32,440 --> 00:24:33,200 IMPORTANT PRIORITY FOR ALS 472 00:24:33,200 --> 00:24:33,760 INVESTIGATIONS AND RESEARCH, 473 00:24:33,760 --> 00:24:38,840 NEXT SLIDE PLEASE. I'M GONNA 474 00:24:38,840 --> 00:24:39,600 TAKE THE LIBERTY OF GOING 475 00:24:39,600 --> 00:24:40,760 THROUGH VERY BRIEFLY THE NEXT 476 00:24:40,760 --> 00:24:41,320 FEW MINUTES OF THE COMMON 477 00:24:41,320 --> 00:24:44,440 ELEMENTS THAT MOTIVATED OUR 478 00:24:44,440 --> 00:24:45,600 THINKING OF THESE ELEMENTS IN 479 00:24:45,600 --> 00:24:47,520 OUR OVERALL PROGRAM. I WANT 480 00:24:47,520 --> 00:24:50,480 TO BEGIN A WORD ABOUT ALS 481 00:24:50,480 --> 00:24:51,440 ITSELF. WE DO KNOW THIS IS A 482 00:24:51,440 --> 00:24:55,560 DISEASE AND THE BRAIN MORE 483 00:24:55,560 --> 00:24:57,000 NEURONS AND WE KNOW THAT IT 484 00:24:57,000 --> 00:24:59,080 STARTS INSIDIOUSLY WITH VOCAL 485 00:24:59,080 --> 00:25:00,560 PARALYSIS WHICH THEN SPREADS 486 00:25:00,560 --> 00:25:02,480 WIDELY. NOW, THE IMPLICATION 487 00:25:02,480 --> 00:25:04,440 OF THAT IS THAT IS 488 00:25:04,440 --> 00:25:04,920 REGRETTABLY, A LONG TIME 489 00:25:04,920 --> 00:25:06,920 BEFORE PEOPLE HAVE AN 490 00:25:06,920 --> 00:25:09,120 ESTABLISHED DIAGNOSIS. THE 491 00:25:09,120 --> 00:25:11,120 TIME. NOW IS UPWARDS OF ONE 492 00:25:11,120 --> 00:25:11,720 YEAR IN THE UNITED STATES AND 493 00:25:11,720 --> 00:25:14,240 IN EUROPE. THIS IS ONE OF THE 494 00:25:14,240 --> 00:25:16,240 ISSUES THAT WE HAVE TO ADDRESS 495 00:25:16,240 --> 00:25:16,960 TO MAKE THE DIAGNOSIS OR 496 00:25:16,960 --> 00:25:19,960 RAPID. IT'S ALSO THE CASE 497 00:25:19,960 --> 00:25:22,160 THAT SOME CASES ARE ASSOCIATED 498 00:25:22,160 --> 00:25:24,840 WITH ATYPICAL FRONTAL TEMPORAL 499 00:25:24,840 --> 00:25:25,480 DEMENTIA THAT WILL COME UP 500 00:25:25,480 --> 00:25:29,400 DURING THE DAY. NEXT SLIDE 501 00:25:29,400 --> 00:25:30,840 PLEASE. THE STRIKING 502 00:25:30,840 --> 00:25:31,440 PATHOLOGY IN THIS DISEASE AS 503 00:25:31,440 --> 00:25:33,360 WE ALL KNOW IS THE LOSS OF THE 504 00:25:33,360 --> 00:25:37,440 MOTOR NEURONS AND AS MOTOR 505 00:25:37,440 --> 00:25:38,880 NEURONS UNPLUG THERE IS MUSCLE 506 00:25:38,880 --> 00:25:40,600 WASTING AND WEAKNESS, AS SHOWN 507 00:25:40,600 --> 00:25:42,760 IN THE TOP FIGURES HERE. 508 00:25:42,760 --> 00:25:44,800 YOU'VE HEARD THE DOCTOR 509 00:25:44,800 --> 00:25:47,120 MENTIONED THE KEY ELEMENTS OF 510 00:25:47,120 --> 00:25:51,080 VIRTUALLY ALL KEYS OF ALS IS A 511 00:25:51,080 --> 00:25:53,080 DYSFUNCTION OF ALL PROTEINS 512 00:25:53,080 --> 00:25:53,720 AND VARIOUS TYPES WHICH ARE 513 00:25:53,720 --> 00:25:57,360 MIS- LOCALIZED TO WREAK HAVOC. 514 00:25:57,360 --> 00:25:59,920 AS WELL AS A POINT THAT WERE 515 00:25:59,920 --> 00:26:01,040 BEGINNING TO UNDERSTAND THAT 516 00:26:01,040 --> 00:26:02,040 THESE CELLS THAT ARE ACTIVATED 517 00:26:02,040 --> 00:26:03,120 IN THIS DISEASE AND IN A SENSE 518 00:26:03,120 --> 00:26:05,960 AS IT GOES IT TAKES A VILLAGE 519 00:26:05,960 --> 00:26:10,440 TO KILL A MOTOR NEURON AND 520 00:26:10,440 --> 00:26:12,440 DISCUSSES WHAT WE CALL MICRO- 521 00:26:12,440 --> 00:26:16,520 NARRATION WHICH IS PART OF THE 522 00:26:16,520 --> 00:26:18,400 PROCESS. I'M GOING TO SIMPLY 523 00:26:18,400 --> 00:26:19,600 REITERATE WHAT THE DOCTOR SAID 524 00:26:19,600 --> 00:26:21,800 WHICH IS THE HUGE ELEPHANT IN 525 00:26:21,800 --> 00:26:22,480 THE ROOM. EVERY TIME YOU HAVE 526 00:26:22,480 --> 00:26:26,920 AN ALS DISCUSSION IT IS SO 527 00:26:26,920 --> 00:26:27,920 SPORADIC, NON- FAMILIAL ALS. 528 00:26:27,920 --> 00:26:29,920 WHAT CAUSES IT? WE DON'T KNOW 529 00:26:29,920 --> 00:26:31,560 BUT WE HAVE IDEAS. WE DO TO 530 00:26:31,560 --> 00:26:33,880 THE 10% OF CASES ARE FAMILIAL 531 00:26:33,880 --> 00:26:39,120 AND SO BECAUSE OF THAT IN THE 532 00:26:39,120 --> 00:26:40,280 EVOLUTION OF GENETICS IT'S 533 00:26:40,280 --> 00:26:41,480 IMPOSSIBLE TO IDENTIFY MANY 534 00:26:41,480 --> 00:26:44,560 ALS GENES. THESE HAVE BEEN 535 00:26:44,560 --> 00:26:45,280 NICELY SUMMARIZED BY MANY 536 00:26:45,280 --> 00:26:46,240 PEOPLE, INCLUDING DOCTOR HARMS 537 00:26:46,240 --> 00:26:47,720 IN THE FIGURE IN THE REFERENCE 538 00:26:47,720 --> 00:26:48,720 ON THE RIGHT. HERE'S THE KEY 539 00:26:48,720 --> 00:26:50,840 POINT. WE ARE ALREADY 540 00:26:50,840 --> 00:26:54,960 LEARNING THAT SOME OF THE 541 00:26:54,960 --> 00:26:56,840 GENES THAT CAUSE FAMILIAL ALS 542 00:26:56,840 --> 00:26:57,360 ARE ALSO IMPLICATED IN 543 00:26:57,360 --> 00:27:01,480 SPORADIC ALS. NEXT SLIDE. 544 00:27:01,480 --> 00:27:02,160 BUT ARE ALSO LEARNING IS THAT 545 00:27:02,160 --> 00:27:06,040 WE ARE CAREFULLY ANALYZING DUE 546 00:27:06,040 --> 00:27:08,200 TO MOTOR NEURONS IN 547 00:27:08,200 --> 00:27:09,640 SURROUNDING CELLS WE FIND THAT 548 00:27:09,640 --> 00:27:13,000 IT'S INCREDIBLY COMPLEX. YOU 549 00:27:13,000 --> 00:27:17,600 CAN SEE THIS SHOWN HERE IN 550 00:27:17,600 --> 00:27:18,720 YELLOW WHICH IS PURPLE GOING 551 00:27:18,720 --> 00:27:19,560 TO THE MUSCLE ON THE RIGHT. 552 00:27:19,560 --> 00:27:22,880 LOOK AT IT CAREFULLY AND NO 553 00:27:22,880 --> 00:27:24,320 MATTER WHAT YOU'RE LOOKING AT 554 00:27:24,320 --> 00:27:30,080 IT WILL BE ABNORMAL IN ALS. 555 00:27:30,080 --> 00:27:31,120 THIS IS DOWNSTREAM * 556 00:27:31,120 --> 00:27:32,360 DOWNSTREAM, DEVASTATING, 557 00:27:32,360 --> 00:27:32,920 PATHOLOGICAL EVENTS THAT ARE 558 00:27:32,920 --> 00:27:33,360 VERY IMPORTANT IN 559 00:27:33,360 --> 00:27:33,960 UNDERSTANDING THIS DISEASE AND 560 00:27:33,960 --> 00:27:37,600 ON THE UPSIDE PROVIDE 561 00:27:37,600 --> 00:27:38,120 POTENTIAL POTENTIAL 562 00:27:38,120 --> 00:27:42,240 INTERVENTION. NEXT SLIDE. I 563 00:27:42,240 --> 00:27:46,040 WANT TO EMPHASIZE TWO-POINT AS 564 00:27:46,040 --> 00:27:47,320 WALTER SAID, IT'S A BIT 565 00:27:47,320 --> 00:27:47,880 DISCOURAGING PERHAPS WE ARE 566 00:27:47,880 --> 00:27:51,240 NOT FURTHER ALONG IN THE 567 00:27:51,240 --> 00:27:53,800 PATHWAY TO THERAPY. THERE ARE 568 00:27:53,800 --> 00:27:55,080 FOUR APPROVED DRUGS, UNTIL 569 00:27:55,080 --> 00:27:56,840 RECENTLY ARE APPROVED. AND 570 00:27:56,840 --> 00:27:58,120 THAT IS PERHAPS NOT AS GOOD AS 571 00:27:58,120 --> 00:28:00,320 WE WOULD LIKE. BUT WHAT I 572 00:28:00,320 --> 00:28:01,760 WOULD LIKE TO EMPHASIZE ON THE 573 00:28:01,760 --> 00:28:05,840 UPSIDE IS THAT THERE IS A HUGE 574 00:28:05,840 --> 00:28:06,440 PIPELINE NOW OF POTENTIAL ALS 575 00:28:06,440 --> 00:28:07,240 THERAPIES INVOLVING, NOT JUST 576 00:28:07,240 --> 00:28:11,600 CONVENTIONAL DRUGS, BUT ALSO 577 00:28:11,600 --> 00:28:12,800 BIOLOGICAL REAGENTS, 578 00:28:12,800 --> 00:28:16,840 ANTIBODIES AND AS WALTER SAID, 579 00:28:16,840 --> 00:28:18,640 THE NUCLEOTIDES AND CELL 580 00:28:18,640 --> 00:28:20,200 THERAPIES AS WELL. BUT MORE 581 00:28:20,200 --> 00:28:20,840 IMPORTANTLY OR AS IMPORTANTLY, 582 00:28:20,840 --> 00:28:21,440 THERE ARE SOME NEW MODELS FOR 583 00:28:21,440 --> 00:28:27,040 HOW TO DO DRUG TRIALS. I 584 00:28:27,040 --> 00:28:29,120 THINK IT WAS AN APPROPRIATE 585 00:28:29,120 --> 00:28:29,640 NOTICE TO UNDERTAKE THIS 586 00:28:29,640 --> 00:28:30,280 ORGANIZATION AS WERE NOT GOING 587 00:28:30,280 --> 00:28:40,680 TO DO THIS OVER THE NEXT 588 00:34:09,000 --> 00:34:12,280 TRANSLATE THE BASIC SENSITIVE 589 00:34:12,280 --> 00:34:14,400 CLINIC. THIS REQUIRES THAT WE 590 00:34:14,400 --> 00:34:16,480 ALSO HAVE A BETTER 591 00:34:16,480 --> 00:34:17,600 INFRASTRUCTURE. THERE'S A LOT 592 00:34:17,600 --> 00:34:18,320 OF EFFORTS IN COLLECTING BIO 593 00:34:18,320 --> 00:34:19,120 SAMPLES FROM PEOPLE LIVING 594 00:34:19,120 --> 00:34:24,680 WITH ALS. THEIR SPINAL FLUID, 595 00:34:24,680 --> 00:34:27,120 BLOOD, THE LOT IS BIO SAMPLES 596 00:34:27,120 --> 00:34:28,520 REALLY ALLOW THE RESEARCHERS 597 00:34:28,520 --> 00:34:29,560 TO BETTER UNDERSTAND THE 598 00:34:29,560 --> 00:34:30,720 DISEASE. THIS SHOULD BE 599 00:34:30,720 --> 00:34:31,920 STRUCTURED IN A MORE ORGANIZED 600 00:34:31,920 --> 00:34:33,800 WAY AND THIS IS ONE OF THE 601 00:34:33,800 --> 00:34:34,360 PRIORITIES OF THIS GROUP HAS 602 00:34:34,360 --> 00:34:40,280 COME UP WITH. AND FINALLY IT 603 00:34:40,280 --> 00:34:42,560 WOULD BE IMPORTANT TO GET MORE 604 00:34:42,560 --> 00:34:44,680 STRUCTURED SUPPORT IN ORDER TO 605 00:34:44,680 --> 00:34:45,640 UTILIZE ALL OF THE INFORMATION 606 00:34:45,640 --> 00:34:47,120 TO REALLY ENABLE THE TRIALS 607 00:34:47,120 --> 00:34:49,760 THAT WE EVENTUALLY WANT TO GET 608 00:34:49,760 --> 00:34:51,600 TO. AND AGAIN, TO FOSTER THE 609 00:34:51,600 --> 00:34:52,200 COLLABORATION BETWEEN INDUSTRY 610 00:34:52,200 --> 00:34:56,360 AND ACADEMIC INSTITUTIONS 611 00:34:56,360 --> 00:34:57,280 WHICH REALLY MAKE THIS 612 00:34:57,280 --> 00:34:58,240 TRANSLATION HAPPENED TO THEN 613 00:34:58,240 --> 00:34:58,760 MOVE FORWARD TO GO INTO 614 00:34:58,760 --> 00:35:01,920 CLINICAL TRIALS. THIS IS OUR 615 00:35:01,920 --> 00:35:02,880 THIRD WORKING GROUP OF THE DAY 616 00:35:02,880 --> 00:35:06,920 IF I COULD HAVE THE NEXT 617 00:35:06,920 --> 00:35:10,920 SLIDE. THIS IS THE GROUP 618 00:35:10,920 --> 00:35:17,840 TRYING TO THIS GROUP IS 619 00:35:17,840 --> 00:35:22,560 CHAIRED BY TIM AND THE 620 00:35:22,560 --> 00:35:23,880 PRIORITIES OF CANADA'S GROUP 621 00:35:23,880 --> 00:35:25,280 THAT ARE REALLY DEFINED TO GET 622 00:35:25,280 --> 00:35:28,200 A BETTER IDEA OF ALS NATIONAL 623 00:35:28,200 --> 00:35:29,480 HISTORY AND THIS WAS BROUGHT 624 00:35:29,480 --> 00:35:31,480 UP BY WALTER EARLIER IN HIS 625 00:35:31,480 --> 00:35:32,320 INTRODUCTION. WE SEE THIS IS 626 00:35:32,320 --> 00:35:34,320 A VERY IMPORTANT TOPIC OF HOW 627 00:35:34,320 --> 00:35:35,680 WE CAN BETTER RUN CLINICAL 628 00:35:35,680 --> 00:35:37,040 TRIALS IF WE HAVE THIS 629 00:35:37,040 --> 00:35:39,520 INFORMATION THAT WE COULD 630 00:35:39,520 --> 00:35:40,280 GATHER THROUGH THESE NATIONAL 631 00:35:40,280 --> 00:35:41,160 HISTORY STUDIES? OF COURSE, 632 00:35:41,160 --> 00:35:43,520 WE WANT TO MAKE SURE THAT WE 633 00:35:43,520 --> 00:35:44,600 HAVE TREATMENTS FOR PEOPLE 634 00:35:44,600 --> 00:35:45,560 LIVING WITH ALS AT ALL STAGES 635 00:35:45,560 --> 00:35:50,920 ACROSS THE DISEASE SPECTRUM. 636 00:35:50,920 --> 00:35:51,960 FOR THOSE THAT ARE 637 00:35:51,960 --> 00:35:52,560 PRESYMPTOMATIC AND POTENTIALLY 638 00:35:52,560 --> 00:35:55,120 GENETIC MUTATION CARRIERS. 639 00:35:55,120 --> 00:35:56,800 FOR THOSE THAT HAD JUST 640 00:35:56,800 --> 00:35:58,200 DEVELOPED THE DISEASE AND FOR 641 00:35:58,200 --> 00:35:59,640 THOSE THAT ARE WAY INTO THE 642 00:35:59,640 --> 00:36:00,400 DISEASE WE HAD TO FIGURE OUT 643 00:36:00,400 --> 00:36:04,320 WAYS THAT WE CAN TARGET ALL OF 644 00:36:04,320 --> 00:36:05,000 HIS PATIENT POPULATIONS AND 645 00:36:05,000 --> 00:36:05,560 THAT WE FIND TREATMENTS THAT 646 00:36:05,560 --> 00:36:09,400 ARE SPECIFICALLY SUITABLE FOR 647 00:36:09,400 --> 00:36:10,640 NO MATTER WHICH STAGE OF THE 648 00:36:10,640 --> 00:36:12,320 DISEASE YOU ARE AT WE WILL 649 00:36:12,320 --> 00:36:14,320 HAVE A TREATMENT FOR YOU. WE 650 00:36:14,320 --> 00:36:16,000 HAVE TO KEEP THAT IN MIND. 651 00:36:16,000 --> 00:36:19,120 AND WITH THAT IN MIND IT IS 652 00:36:19,120 --> 00:36:20,760 ALSO THIRDLY IMPORTANT THAT WE 653 00:36:20,760 --> 00:36:22,560 DEFINED HOW DOES ALS MANIFEST 654 00:36:22,560 --> 00:36:26,280 EARLY ON? IT IS A START 655 00:36:26,280 --> 00:36:27,400 TEN-YEARS PRIOR TO DISEASE 656 00:36:27,400 --> 00:36:30,760 ONSET? 20-YEARS PRIOR TO 657 00:36:30,760 --> 00:36:33,440 THAT? ARE THERE WAYS FOR US 658 00:36:33,440 --> 00:36:34,920 TO UNDERSTAND HOW THAT 659 00:36:34,920 --> 00:36:36,800 HAPPENS? THIS IS AN IMPORTANT 660 00:36:36,800 --> 00:36:37,560 PRIORITY FOR US TO BETTER 661 00:36:37,560 --> 00:36:38,040 UNDERSTAND WHEN COULD 662 00:36:38,040 --> 00:36:39,840 POTENTIALLY THERAPEUTIC 663 00:36:39,840 --> 00:36:41,040 INTERVENTION WITH PATIENTS AT 664 00:36:41,040 --> 00:36:42,120 THE EARLY SIGNS OF THE 665 00:36:42,120 --> 00:36:42,680 DISEASE, EVEN THOUGH THERE'S 666 00:36:42,680 --> 00:36:46,440 NO OBVIOUS SYMPTOMS YET 667 00:36:46,440 --> 00:36:48,280 VISIBLE WITH PEOPLE LIVING 668 00:36:48,280 --> 00:36:50,120 WITH THIS DISEASE. THESE ARE 669 00:36:50,120 --> 00:36:52,400 THE MAJOR THREE TOPICS THAT 670 00:36:52,400 --> 00:36:54,960 WERE GOING TO DISCUSS TODAY. 671 00:36:54,960 --> 00:36:55,560 IT'S INTERESTING AND IMPORTANT 672 00:36:55,560 --> 00:36:56,160 TOPIC THAT WE ALSO APPRECIATE 673 00:36:56,160 --> 00:37:01,160 WAS PUT TOGETHER FROM THE 674 00:37:01,160 --> 00:37:02,640 COMMITTEE MEMBERS THAT EITHER 675 00:37:02,640 --> 00:37:04,800 LIVE WITH ALS OR HAVE TAKEN 676 00:37:04,800 --> 00:37:06,640 CARE OF PATIENTS WITH ALS IS 677 00:37:06,640 --> 00:37:08,920 THE NEXT SESSION. AND IF I 678 00:37:08,920 --> 00:37:09,480 COULD HAVE THE NEXT SLIDE. 679 00:37:09,480 --> 00:37:13,040 THIS IS REALLY WHAT WE CALL 680 00:37:13,040 --> 00:37:14,920 THE LIVED EXPERIENCE TO PANEL. 681 00:37:14,920 --> 00:37:16,280 THIS WAS SUPPORTED STRONGLY BY 682 00:37:16,280 --> 00:37:16,800 THE CHEERS OF THIS PANEL, 683 00:37:16,800 --> 00:37:20,520 WHICH IS LANE AND ELLEN 684 00:37:20,520 --> 00:37:21,480 PHILLIPS WHO THOUGHT THIS 685 00:37:21,480 --> 00:37:23,920 WOULD BE AN IMPORTANT PART OF 686 00:37:23,920 --> 00:37:25,720 THIS WORKSHOP IN ORDER FOR US 687 00:37:25,720 --> 00:37:27,040 TO BETTER UNDERSTAND WHY ARE 688 00:37:27,040 --> 00:37:30,560 WE DOING THIS? WHY ARE WE 689 00:37:30,560 --> 00:37:32,160 SITTING BEHIND OUR DESKS AND 690 00:37:32,160 --> 00:37:33,920 TRYING TO UNDERSTAND HOW WE 691 00:37:33,920 --> 00:37:34,680 CAN TACKLE THIS DISEASE? WE 692 00:37:34,680 --> 00:37:36,400 NEED TO HEAR FROM THE PEOPLE 693 00:37:36,400 --> 00:37:37,600 WHO HAVE LIVED WITH THE 694 00:37:37,600 --> 00:37:38,160 DISEASE AND THE PEOPLE WHO 695 00:37:38,160 --> 00:37:39,160 TAKE CARE OF THE PEOPLE THAT 696 00:37:39,160 --> 00:37:44,280 HAVE THIS DISEASE. I REALLY 697 00:37:44,280 --> 00:37:44,840 APPRECIATE LANE AND ELLEN IT 698 00:37:44,840 --> 00:37:48,080 IS EFFORTS WITH THIS AND I 699 00:37:48,080 --> 00:37:49,760 WOULD LIKE TO INTRODUCE OUR 700 00:37:49,760 --> 00:37:50,320 TWO COMMITTEE MEMBERS. THEY 701 00:37:50,320 --> 00:37:52,880 PUT THIS TOGETHER AND I HOPE 702 00:37:52,880 --> 00:37:53,480 AND LOOK FOR MORE FORWARD TO 703 00:37:53,480 --> 00:37:59,280 THE PANEL DISCUSSION. 704 00:37:59,280 --> 00:38:02,520 >>: GOOD MORNING 705 00:38:02,520 --> 00:38:03,080 EVERYONE. COULD YOU PUT THE 706 00:38:03,080 --> 00:38:05,920 FIRST SLIDE UP? THE TITLE 707 00:38:05,920 --> 00:38:10,280 SLIDE PLEASE, THANK YOU. GOOD 708 00:38:10,280 --> 00:38:12,400 MORNING TO EVERYBODY OUT 709 00:38:12,400 --> 00:38:15,240 THERE! AS YOU CAN TELL I HAVE 710 00:38:15,240 --> 00:38:20,120 ALS, EVEN HERE IN MY SPEECH. 711 00:38:20,120 --> 00:38:22,240 WHAT WE WANT TO DO THIS 712 00:38:22,240 --> 00:38:27,120 MORNING IS GIVE YOU INSIGHT 713 00:38:27,120 --> 00:38:32,120 INTO THE WORLD AS I WOULD CALL 714 00:38:32,120 --> 00:38:33,840 IT. I CALL THIS AN ALS 715 00:38:33,840 --> 00:38:35,760 EXPERIENCE EXPERTS. THOSE 716 00:38:35,760 --> 00:38:40,440 PEOPLE WITH ALS, ALS 717 00:38:40,440 --> 00:38:40,960 CAREGIVERS AND ALS GENE 718 00:38:40,960 --> 00:38:44,600 CARRIERS HAVE HAD AND CONTINUE 719 00:38:44,600 --> 00:38:51,880 TO HAVE INVOLVEMENT IN THE ALS 720 00:38:51,880 --> 00:38:52,440 STRATEGIC PLANNING PROCESS. 721 00:38:52,440 --> 00:38:56,240 AS RITA MENTIONED, THIS 722 00:38:56,240 --> 00:38:58,840 SESSION WILL BE MODERATED BY 723 00:38:58,840 --> 00:39:03,720 MYSELF. IT ALSO ELLEN. CAN 724 00:39:03,720 --> 00:39:04,520 YOU PUT UP THE FIRST SLIDE 725 00:39:04,520 --> 00:39:08,760 PLEASE. NEXT SLIDE. THIS IS 726 00:39:08,760 --> 00:39:17,200 OUR PANEL. AS YOU CAN SEE, 727 00:39:17,200 --> 00:39:18,200 THEY ARE NINE, ALS EXPERIENCE 728 00:39:18,200 --> 00:39:22,040 EXPERTS ON THIS PANEL. AND 729 00:39:22,040 --> 00:39:22,600 INDEED THERE ARE TWO OTHER 730 00:39:22,600 --> 00:39:27,120 PEOPLE WHO ARE ALS EXPERIENCED 731 00:39:27,120 --> 00:39:28,600 EXPERTS WHO ARE INVOLVED IN 732 00:39:28,600 --> 00:39:31,440 THE PLANNING PROCESS. THEY 733 00:39:31,440 --> 00:39:33,400 ARE BOTH COCHAIRS OF 734 00:39:33,400 --> 00:39:34,520 COMMITTEES AND WILL BE 735 00:39:34,520 --> 00:39:39,840 SPEAKING LATER. SO, A TOTAL 736 00:39:39,840 --> 00:39:50,280 OF 11 OUT OF 52, 52 BEING 737 00:39:54,400 --> 00:39:54,960 NONPERSONNEL BUT 11 WERE 738 00:39:54,960 --> 00:39:58,840 PEOPLE WITH ALS EXPERIENCED 739 00:39:58,840 --> 00:40:04,280 EXPERTS. THAT IS 740 00:40:04,280 --> 00:40:07,960 UNPRECEDENTED WITH A FEDERAL 741 00:40:07,960 --> 00:40:12,960 AGENCY BY DEVELOPING A 742 00:40:12,960 --> 00:40:14,880 STRATEGIC PLAN. WE CAN TALK 743 00:40:14,880 --> 00:40:19,080 MORE ABOUT THAT LATER TODAY OR 744 00:40:19,080 --> 00:40:21,560 TOMORROW. WE DO HAVE A SECOND 745 00:40:21,560 --> 00:40:23,920 PANEL TOMORROW. WITH THAT, 746 00:40:23,920 --> 00:40:27,560 LET ME GIVE YOU A 22nd 747 00:40:27,560 --> 00:40:30,400 BACKGROUND OF MY ALS. I WAS 748 00:40:30,400 --> 00:40:34,640 INITIALLY DIAGNOSED WITH P ALS 749 00:40:34,640 --> 00:40:38,400 IN 2017. THAT WAS CHANGED TO 750 00:40:38,400 --> 00:40:42,840 ALS IN NOVEMBER OF 2020. MY 751 00:40:42,840 --> 00:40:43,400 TRAINING PHYSICIAN, HIS NAME 752 00:40:43,400 --> 00:40:45,760 WAS ALREADY MENTIONED THIS 753 00:40:45,760 --> 00:40:48,480 MORNING, DOCTOR MATTHEW HARMS 754 00:40:48,480 --> 00:40:53,560 AT COLUMBIA UNIVERSITY. WITH 755 00:40:53,560 --> 00:40:54,680 THAT I'M GOING TO TURN IT OVER 756 00:40:54,680 --> 00:40:58,600 TO ELLEN TO COORDINATE THE 757 00:40:58,600 --> 00:41:00,160 INTRODUCTIONS OF OUR PANEL AND 758 00:41:00,160 --> 00:41:04,440 THE BACKGROUND AND HERSELF. 759 00:41:04,440 --> 00:41:05,400 >>: THANK YOU IT'S BEEN A 760 00:41:05,400 --> 00:41:05,960 PLEASURE WORKING WITH YOU ON 761 00:41:05,960 --> 00:41:11,320 THIS PROJECT. YOU HAVE A LOT 762 00:41:11,320 --> 00:41:12,200 OF ENERGY AND YOU'RE CERTAINLY 763 00:41:12,200 --> 00:41:13,800 SMART AND I LOOK AT YOU AS A 764 00:41:13,800 --> 00:41:15,120 MENTOR FOR WHAT WE HAVE DONE 765 00:41:15,120 --> 00:41:20,160 HERE TODAY. MY NAME AS YOU 766 00:41:20,160 --> 00:41:28,040 HEARD. CURRENTLY PHILADELPHIA 767 00:41:28,040 --> 00:41:30,960 ALS ASSOCIATION I'M ALSO A 768 00:41:30,960 --> 00:41:32,360 FORMER TRUSTEE OF THE NATIONAL 769 00:41:32,360 --> 00:41:34,920 ALS ASSOCIATION. I CONSIDER 770 00:41:34,920 --> 00:41:36,440 MYSELF A PATIENT ADVOCATE 771 00:41:36,440 --> 00:41:38,520 HAVING LOST MY HUSBAND ALAN TO 772 00:41:38,520 --> 00:41:39,840 ALS IN 1980 FOUR, WHICH SEEMS 773 00:41:39,840 --> 00:41:46,480 LIKE THE DARK AGES. HE WAS 36 774 00:41:46,480 --> 00:41:47,760 AND OUR DAUGHTER AMY WAS THREE 775 00:41:47,760 --> 00:41:51,880 AT THE TIME. OUR NEUROLOGISTS 776 00:41:51,880 --> 00:41:52,760 WAS DOCTOR KENNETH FISCHBECK, 777 00:41:52,760 --> 00:41:56,920 YOU MAY RECOGNIZE THAT NAME. 778 00:41:56,920 --> 00:41:59,560 I LEARNED MORE ABOUT ALS OVER 779 00:41:59,560 --> 00:42:00,400 THE YEARS BY HAVING THAT 780 00:42:00,400 --> 00:42:04,120 PRIVILEGE OF SERVING ON THE NI 781 00:42:04,120 --> 00:42:07,160 NDS COUNCIL, THE FDA ADVISORY 782 00:42:07,160 --> 00:42:09,560 COMMITTEE ON CENTRAL AND 783 00:42:09,560 --> 00:42:11,400 PERIPHERAL NERVOUS DISORDERS, 784 00:42:11,400 --> 00:42:12,320 AND THE INAUGURAL DEPARTMENT 785 00:42:12,320 --> 00:42:17,280 OF DEFENSE ALS RESEARCH GROUP. 786 00:42:17,280 --> 00:42:20,000 NOW I SPEND MY TIME IT IS 787 00:42:20,000 --> 00:42:20,960 FUNDRAISING FOR OUR CHAPTER 788 00:42:20,960 --> 00:42:21,920 WHICH INCLUDES WORKING WITH 789 00:42:21,920 --> 00:42:24,520 THE PHILADELPHIA PHILLIES. I 790 00:42:24,520 --> 00:42:26,000 REFRAINED FROM WEARING MY 791 00:42:26,000 --> 00:42:28,760 JERSEY. THEY HAVE RAISED OVER 792 00:42:28,760 --> 00:42:29,360 $20 MILLION FOR PATIENT CARE 793 00:42:29,360 --> 00:42:35,240 AND RESEARCH. A WORLD SERIES 794 00:42:35,240 --> 00:42:35,840 CHAMPIONSHIP THIS IS INCREASE 795 00:42:35,840 --> 00:42:39,760 THAT AMOUNT SIGNIFICANTLY. I 796 00:42:39,760 --> 00:42:41,920 WOULD LIKE TO INTRODUCE OUR 797 00:42:41,920 --> 00:42:47,720 PANEL AND THEY WILL INTRODUCE 798 00:42:47,720 --> 00:42:51,240 THEMSELVES. BUT I WILL CALL 799 00:42:51,240 --> 00:43:01,640 ON THEM. GWEN PETERSON. 800 00:43:02,240 --> 00:43:03,760 >>: HELLO. MY NAME IS 801 00:43:03,760 --> 00:43:05,480 GWEN PETERSON. I WAS 802 00:43:05,480 --> 00:43:10,440 DIAGNOSED AT 30 TWO-YEARS OLD 803 00:43:10,440 --> 00:43:11,040 IN 2018 AND IT'S REALLY GOOD 804 00:43:11,040 --> 00:43:20,600 TO BE HERE, THANK YOU. 805 00:43:20,600 --> 00:43:22,760 >>: ED RAPA. 806 00:43:22,760 --> 00:43:25,280 >>: THANK YOU. MY NAME 807 00:43:25,280 --> 00:43:26,800 IS ED AND I AM THE FORMER 808 00:43:26,800 --> 00:43:27,360 CATERPILLAR GROUP PRESIDENT 809 00:43:27,360 --> 00:43:30,560 AND ANNOUNCED MY RETIREMENT AT 810 00:43:30,560 --> 00:43:32,720 THE END OF 2015 WHEN I WAS 811 00:43:32,720 --> 00:43:36,520 DIAGNOSED DIAGNOSED WITH ALS. 812 00:43:36,520 --> 00:43:39,520 IN THE PALS TOLD ME YOU GOT A 813 00:43:39,520 --> 00:43:40,120 CHOICE YOU CAN STAY BETTER AND 814 00:43:40,120 --> 00:43:42,160 YOU CAN STAY ENGAGED I AM 815 00:43:42,160 --> 00:43:48,920 OPTING FOR THE LATTER. AND 816 00:43:48,920 --> 00:43:52,640 THIS IS WHERE WE BRING 817 00:43:52,640 --> 00:43:53,320 TOGETHER FOR ALS LARGEST 818 00:43:53,320 --> 00:43:54,880 REPOSITORY DATA AND IPS CELLS 819 00:43:54,880 --> 00:43:56,640 GATHERED IN THE HISTORY OF THE 820 00:43:56,640 --> 00:43:58,200 DISEASE AND THEY'RE PUTTING ON 821 00:43:58,200 --> 00:43:59,040 AN OPEN-SOURCE PLATFORM AND 822 00:43:59,040 --> 00:43:59,520 MAKE IT AVAILABLE TO 823 00:43:59,520 --> 00:44:00,120 RESEARCHERS AROUND THE WORLD 824 00:44:00,120 --> 00:44:05,920 FOR FREE. NO, IT'S NOT 825 00:44:05,920 --> 00:44:06,440 SELLING TRACTORS BUT IT'S 826 00:44:06,440 --> 00:44:08,160 MEANINGFUL WORK AS IS 827 00:44:08,160 --> 00:44:10,440 PARTICIPATION IN THE NIH 828 00:44:10,440 --> 00:44:15,120 EFFORT THAT WERE DISCUSSING 829 00:44:15,120 --> 00:44:25,320 TODAY. THANK YOU, COOPER. 830 00:44:25,320 --> 00:44:31,080 >>: COOPER [INDISTINCT] 831 00:44:31,080 --> 00:44:33,920 SORRY WELL, MY TOTALLY BLUE. 832 00:44:33,920 --> 00:44:36,320 I GUESS THE BACKGROUND DIDN'T 833 00:44:36,320 --> 00:44:41,360 WORK FOR ME OR SOMETHING. 834 00:44:41,360 --> 00:44:43,200 MAYBE I SHOULD TURN OFF MY 835 00:44:43,200 --> 00:44:44,560 CAMERA SO LET'S NOT 836 00:44:44,560 --> 00:44:45,240 DISTRACTING. MY NAME IS 837 00:44:45,240 --> 00:44:48,920 COOPER PENNER, I AM A CURRENT 838 00:44:48,920 --> 00:44:49,520 PHD STUDENT AT THE UNIVERSITY 839 00:44:49,520 --> 00:44:52,600 OF PENNSYLVANIA AND I WORK 840 00:44:52,600 --> 00:44:56,040 WITH IP SC DERIVED NEURONS TO 841 00:44:56,040 --> 00:44:56,480 BASICALLY STUDY THE 842 00:44:56,480 --> 00:44:57,440 PATHOGENESIS OF ALS AND I WORK 843 00:44:57,440 --> 00:44:59,480 IN THE CLINIC EVERY WEEK 844 00:44:59,480 --> 00:45:03,000 HELPING FAMILIES NAVIGATE 845 00:45:03,000 --> 00:45:04,960 CAREGIVING. I CARED FOR MY 846 00:45:04,960 --> 00:45:06,560 MOM FOR THREE-YEARS WHILE SHE 847 00:45:06,560 --> 00:45:08,640 WENT THROUGH HER JOURNEY WITH 848 00:45:08,640 --> 00:45:13,360 ALS AND I MYSELF AM A CARRIER. 849 00:45:13,360 --> 00:45:15,120 SO I LIVE WITH THIS DISEASE 850 00:45:15,120 --> 00:45:16,320 EVERY DAY. I'M REALLY 851 00:45:16,320 --> 00:45:18,120 GRATEFUL TO BE HERE ANYMORE 852 00:45:18,120 --> 00:45:19,240 AND GRATEFUL THAT SO MANY 853 00:45:19,240 --> 00:45:19,840 PEOPLE HAVE SIGNED UP AND HAVE 854 00:45:19,840 --> 00:45:22,000 JOINED US. LOOKING FORWARD TO 855 00:45:22,000 --> 00:45:24,680 THE CONVERSATION. 856 00:45:24,680 --> 00:45:31,360 >>: THANK YOU COOPER. 857 00:45:31,360 --> 00:45:31,600 ANDREA. 858 00:45:31,600 --> 00:45:32,360 >>: GOOD MORNING I AM 859 00:45:32,360 --> 00:45:33,920 ANDREA PAUL SPACKMAN AND I 860 00:45:33,920 --> 00:45:38,080 LOST MY MOTHER SALLY TO ALS IN 861 00:45:38,080 --> 00:45:39,400 2010 THREE-YEARS AFTER HER 862 00:45:39,400 --> 00:45:40,520 DIAGNOSIS. I WAS ONE OF HER 863 00:45:40,520 --> 00:45:41,080 PRIMARY CAREGIVERS AND TRULY 864 00:45:41,080 --> 00:45:44,760 THIS WAS A PROFOUND EXPERIENCE 865 00:45:44,760 --> 00:45:45,640 THAT CHANGED MY LIFE IN MANY 866 00:45:45,640 --> 00:45:49,080 WAYS ALSO CHANGE MY CAREER 867 00:45:49,080 --> 00:45:49,840 TRAJECTORY. SHORTLY AFTER SHE 868 00:45:49,840 --> 00:45:51,920 DIED AND LEFT A 30 YEAR CAREER 869 00:45:51,920 --> 00:45:52,760 INVESTMENT IN MANAGEMENT AND 870 00:45:52,760 --> 00:45:55,120 THEN BEGAN TO SERVE AS A BOARD 871 00:45:55,120 --> 00:45:59,600 MEMBER FOR THE ASSOCIATION FOR 872 00:45:59,600 --> 00:46:02,000 A COUPLE OF YEARS AND I CAN AS 873 00:46:02,000 --> 00:46:02,880 CEO OF THE OLDEST INDEPENDENT 874 00:46:02,880 --> 00:46:05,640 ALS GROUP IN THE COUNTRY. I'M 875 00:46:05,640 --> 00:46:10,640 ALSO ON THE BOARD OF DIRECTORS 876 00:46:10,640 --> 00:46:14,240 AND DO LOTS OF OUTREACH FOR 877 00:46:14,240 --> 00:46:18,920 OTHER ORGANIZATIONS. I AM 878 00:46:18,920 --> 00:46:20,920 REALLY HONORED TO BE PART OF 879 00:46:20,920 --> 00:46:21,640 THIS GROUP AND THAT WERE DOING 880 00:46:21,640 --> 00:46:24,120 TODAY. THANK YOU. 881 00:46:24,120 --> 00:46:29,320 >>: THANK YOU ANDREA, 882 00:46:29,320 --> 00:46:29,560 ALLISON. 883 00:46:29,560 --> 00:46:35,760 >>: GOOD MORNING. MY 884 00:46:35,760 --> 00:46:37,920 NAME IS ALLISON I'M SURVIVING 885 00:46:37,920 --> 00:46:39,040 SPOUSE OF ALS HAVING LOST MY 886 00:46:39,040 --> 00:46:42,520 HUSBAND 20-YEARS TO ALS AND 887 00:46:42,520 --> 00:46:43,320 EARLY 2016. AFTER A VERY 888 00:46:43,320 --> 00:46:46,120 QUICK AND DEVASTATING 380 DAY 889 00:46:46,120 --> 00:46:46,680 JOURNEY FROM DIAGNOSIS TO 890 00:46:46,680 --> 00:46:50,240 GAINING HIS WINGS. AS WITH 891 00:46:50,240 --> 00:46:51,720 MANY OF US WHO HAVE 892 00:46:51,720 --> 00:46:52,320 EXPERIENCED THIS UP CLOSE AND 893 00:46:52,320 --> 00:46:54,760 PERSONAL, THE PERSON I WAS 894 00:46:54,760 --> 00:46:59,120 LEFT TO THE PERSON I LOVED. I 895 00:46:59,120 --> 00:46:59,960 HAD A SUCCESSFUL CAREER AND 896 00:46:59,960 --> 00:47:02,280 TECHNOLOGY I CHOSE TO GO BACK 897 00:47:02,280 --> 00:47:03,080 TO SCHOOL AND CITY CLINICAL 898 00:47:03,080 --> 00:47:04,800 RESEARCH ADMINISTRATION. FOR 899 00:47:04,800 --> 00:47:06,440 THE PAST SIX-YEARS OR SO I 900 00:47:06,440 --> 00:47:09,280 FOUND MY HOME AND FOREVER 901 00:47:09,280 --> 00:47:10,400 FAMILY IN THE ALS COMMUNITY. 902 00:47:10,400 --> 00:47:11,000 I WORK AS PART OF THE PATIENT 903 00:47:11,000 --> 00:47:15,120 TEAM AT THE MASSACHUSETTS 904 00:47:15,120 --> 00:47:16,640 GENERAL HOSPITAL AND ICE ON 905 00:47:16,640 --> 00:47:17,680 THE RECURSION AND RETENTION 906 00:47:17,680 --> 00:47:19,680 EXPANDED ACCESS AND PATIENT 907 00:47:19,680 --> 00:47:20,240 ADVISORY COMMITTEES FOR THE 908 00:47:20,240 --> 00:47:23,920 TRIALS. I'M ALSO VERY 909 00:47:23,920 --> 00:47:25,480 INVOLVED WITH THE ALS 910 00:47:25,480 --> 00:47:27,000 CONSORTIUM SERVING AS A 911 00:47:27,000 --> 00:47:28,640 PATIENT LIAISON TO THE 912 00:47:28,640 --> 00:47:30,480 EXECUTIVE LEADERSHIP TEAM, THE 913 00:47:30,480 --> 00:47:31,160 COCHAIR OF THE PATIENT 914 00:47:31,160 --> 00:47:32,240 EDUCATION ADVOCACY COMMITTEE 915 00:47:32,240 --> 00:47:32,720 AND I ALSO SIT ON THE 916 00:47:32,720 --> 00:47:37,600 RETENTION AND RECRUITMENT 917 00:47:37,600 --> 00:47:39,120 COMMITTEES. FINALLY, SERVE AS 918 00:47:39,120 --> 00:47:40,880 A MEMBER OF THE BOARD OF 919 00:47:40,880 --> 00:47:42,920 DIRECTORS AND THE ARIZONA 920 00:47:42,920 --> 00:47:45,040 CHAPTER OF THE ALS 921 00:47:45,040 --> 00:47:46,040 ASSOCIATION. I PARTICIPATED 922 00:47:46,040 --> 00:47:48,240 IN THE CDM RP THAT 923 00:47:48,240 --> 00:47:49,120 CONGRESSIONALLY DIRECTED 924 00:47:49,120 --> 00:47:49,800 MEDICAL RESEARCH PROGRAM AS A 925 00:47:49,800 --> 00:47:51,840 CONSUMER REVIEWER. I WAS PART 926 00:47:51,840 --> 00:47:54,800 OF THE OPPORTUNITIES FOR 927 00:47:54,800 --> 00:47:55,200 COLLABORATIONS AND 928 00:47:55,200 --> 00:47:56,040 PARTNERSHIPS WORK GROUP AND A 929 00:47:56,040 --> 00:47:57,000 VERY HAPPY TO BE HERE WITH A 930 00:47:57,000 --> 00:47:58,080 LOT OF MY FRIENDS ON THE 931 00:47:58,080 --> 00:48:02,120 PANEL. THANK YOU FOR HAVING 932 00:48:02,120 --> 00:48:03,120 ME. 933 00:48:03,120 --> 00:48:04,960 >>: THANK YOU ALLISON. I 934 00:48:04,960 --> 00:48:07,160 DON'T THINK THAT NADIA IS HERE 935 00:48:07,160 --> 00:48:09,360 YET, SHE SENT AN E-MAIL THAT 936 00:48:09,360 --> 00:48:10,920 SHE PROBABLY WILL NOT BE ON 937 00:48:10,920 --> 00:48:12,200 UNTIL 11:00 A.M. I AM 938 00:48:12,200 --> 00:48:14,760 ASSUMING, UNLESS SHE SPEAKS UP 939 00:48:14,760 --> 00:48:18,440 WE WILL GET HER AFTER A LITTLE 940 00:48:18,440 --> 00:48:25,880 LATER IN OUR PORTION. THAT 941 00:48:25,880 --> 00:48:26,760 BRINGS US TO STEVE. 942 00:48:26,760 --> 00:48:28,320 >>: GOOD MORNING 943 00:48:28,320 --> 00:48:29,520 EVERYBODY, MY NAME IS STEVE 944 00:48:29,520 --> 00:48:31,120 KOWALSKI AND I WAS DIAGNOSED 945 00:48:31,120 --> 00:48:34,360 IN AUGUST OF 2017, WITH 946 00:48:34,360 --> 00:48:36,080 SPORADIC ALS. MY FIRST 947 00:48:36,080 --> 00:48:39,200 SYMPTOM WAS THE INABILITY TO 948 00:48:39,200 --> 00:48:41,400 USE MY TOES IN MY RIGHT FOOT 949 00:48:41,400 --> 00:48:43,080 WHICH IS CLASSICALLY KNOWN AS 950 00:48:43,080 --> 00:48:46,640 THE BEGINNING OF DROP FOOT IN 951 00:48:46,640 --> 00:48:47,640 THIS DISEASE. I LIVE IN 952 00:48:47,640 --> 00:48:48,520 BOSTON MASSACHUSETTS WITH 953 00:48:48,520 --> 00:48:49,440 THREE OF MY CHILDREN WHO LIVED 954 00:48:49,440 --> 00:48:50,040 THERE AS WELL AND HELP TAKE 955 00:48:50,040 --> 00:48:53,600 CARE OF ME WITH MAINLY MY 956 00:48:53,600 --> 00:48:58,240 MOBILITY ISSUES. I RETIRED 957 00:48:58,240 --> 00:48:59,200 AFTER 30 FOUR-YEARS IN HIGH 958 00:48:59,200 --> 00:49:02,600 TECH AND I CAN SAY NOW MY 959 00:49:02,600 --> 00:49:04,080 FULL-TIME JOB AS ALS ADVOCACY. 960 00:49:04,080 --> 00:49:04,680 THANK YOU FOR THIS OPPORTUNITY 961 00:49:04,680 --> 00:49:10,760 TO PARTICIPATE. 962 00:49:10,760 --> 00:49:17,200 >>: THANK YOU EVERYONE. 963 00:49:17,200 --> 00:49:21,720 >>: NEXT SLIDE ASLEEP 964 00:49:21,720 --> 00:49:23,200 NEXT SLIDE PLEASE. GO AHEAD. 965 00:49:23,200 --> 00:49:25,440 >>: HERE I GO. THIS IS A 966 00:49:25,440 --> 00:49:28,680 QUESTION FOR EVERYONE AND WE 967 00:49:28,680 --> 00:49:31,760 WILL GO AROUND AGAIN WHAT WERE 968 00:49:31,760 --> 00:49:33,280 YOUR THOUGHTS AND FEELINGS 969 00:49:33,280 --> 00:49:33,920 ABOUT ALS RESEARCH BEFORE? 970 00:49:33,920 --> 00:49:35,960 COMPARE THIS TO AFTER THE 971 00:49:35,960 --> 00:49:40,640 DEVELOPMENT OF THE NIH 972 00:49:40,640 --> 00:49:41,240 BACKSLASH AND I NDS STRATEGIC 973 00:49:41,240 --> 00:49:45,760 PLAN? WE WILL START AGAIN. 974 00:49:45,760 --> 00:49:52,160 >>: ALL RIGHT, SO MY MY 975 00:49:52,160 --> 00:49:52,720 THOUGHTS AND FEELINGS ABOUT 976 00:49:52,720 --> 00:49:59,480 ALS REACH SEARCH THAT COMPARED 977 00:49:59,480 --> 00:50:00,520 AFTER THE DRAFT PLAN REALLY AT 978 00:50:00,520 --> 00:50:07,000 THE MOST BASIC LEVEL HAVE NOT 979 00:50:07,000 --> 00:50:10,120 CHANGED. WE DON'T KNOW A LOT 980 00:50:10,120 --> 00:50:14,960 ABOUT ALS AND CERTAINLY THIS 981 00:50:14,960 --> 00:50:17,520 DRAFT PLAN IS DESPERATELY 982 00:50:17,520 --> 00:50:22,080 TRYING TO GET US THERE. 983 00:50:22,080 --> 00:50:25,800 AGAIN, A BASIC LEVEL THE MORE 984 00:50:25,800 --> 00:50:27,920 WE KNOW ABOUT ALS THE CLOSER 985 00:50:27,920 --> 00:50:34,320 WE ARE GOING TO GET TO HAVING 986 00:50:34,320 --> 00:50:39,080 DISEASE MODIFYING THERAPIES 987 00:50:39,080 --> 00:50:41,320 AND WHAT I MEAN BY THAT ARE 988 00:50:41,320 --> 00:50:43,240 THERAPIES THAT EITHER HALT 989 00:50:43,240 --> 00:50:49,800 PROGRESSION OR REVERSE 990 00:50:49,800 --> 00:50:54,680 SYMPTOMS. TO DO THAT PATIENT 991 00:50:54,680 --> 00:50:55,280 PARTICIPATION IN THE RESEARCH 992 00:50:55,280 --> 00:51:03,560 IS MISSION-CRITICAL I JUST 993 00:51:03,560 --> 00:51:05,040 WANT TO SAY THAT WE CAN HAVE A 994 00:51:05,040 --> 00:51:12,480 BLOCKBUSTER DRUG FOR ALS THEY 995 00:51:12,480 --> 00:51:14,200 HAD THE BEST STUDY DESIGNED IN 996 00:51:14,200 --> 00:51:14,760 THE WORLD BUT IF YOU DON'T 997 00:51:14,760 --> 00:51:18,880 HAVE PATIENTS WHO PARTICIPATE 998 00:51:18,880 --> 00:51:24,360 IT IS SORT OF MOOT. SO, THOSE 999 00:51:24,360 --> 00:51:31,080 ARE MY BASIC THOUGHTS AND I 1000 00:51:31,080 --> 00:51:34,840 WILL STOP THERE. 1001 00:51:34,840 --> 00:51:36,400 >>: THANK YOU. I WILL 1002 00:51:36,400 --> 00:51:38,680 REPEAT THIS FOR EACH SPEAKER, 1003 00:51:38,680 --> 00:51:39,280 WHAT WERE YOUR THOUGHTS AND 1004 00:51:39,280 --> 00:51:41,040 FEELINGS ABOUT ALS RESEARCH 1005 00:51:41,040 --> 00:51:46,880 BEFORE COMPARED TO AFTER THE 1006 00:51:46,880 --> 00:51:50,840 NIGHT H/AND I NDS ALS 1007 00:51:50,840 --> 00:51:52,520 STRATEGIC PLAN? 1008 00:51:52,520 --> 00:51:53,520 >>: I WOULD SAY PRIOR TO 1009 00:51:53,520 --> 00:51:54,520 THE STRATEGIC PLAN I WAS A BIT 1010 00:51:54,520 --> 00:51:56,520 FRUSTRATED BY THE FRAGMENTED 1011 00:51:56,520 --> 00:51:59,240 NATURE OF ALL OF THE EFFORTS 1012 00:51:59,240 --> 00:52:05,520 IN THIS ALS SPACE TAUGHT ME 1013 00:52:05,520 --> 00:52:06,040 THE GREAT INNOVATION WITH 1014 00:52:06,040 --> 00:52:09,160 GREAT COLLABORATION AND WHILE 1015 00:52:09,160 --> 00:52:11,200 THIS POSITIVE EXAMPLES 1016 00:52:11,200 --> 00:52:15,560 HISTORICALLY THIS HAS NOT BEEN 1017 00:52:15,560 --> 00:52:26,040 THE MOST COOPERATIVE FIELD. 1018 00:52:27,840 --> 00:52:28,360 IT REMINDS ME OF RECOVERING 1019 00:52:28,360 --> 00:52:28,960 BUT I WILL REMIND THE GROUP IS 1020 00:52:28,960 --> 00:52:33,720 NOT FOR THE FAINT OF HEART. 1021 00:52:33,720 --> 00:52:34,520 STRATEGY DEVELOPMENT IS 1022 00:52:34,520 --> 00:52:34,960 RELATIVELY BUSY BUT 1023 00:52:34,960 --> 00:52:38,760 IMPLEMENTATION IS THE HARD 1024 00:52:38,760 --> 00:52:42,920 PART. THIS IS NOT WITHOUT 1025 00:52:42,920 --> 00:52:45,840 HOPE THIS OVER THE NEXT FEW 1026 00:52:45,840 --> 00:52:47,320 DAYS OR TO HEAR A LOT ABOUT 1027 00:52:47,320 --> 00:52:48,480 INITIATIVES MAKE A DIFFERENCE 1028 00:52:48,480 --> 00:52:51,200 IN THE DISEASE. THE NIH CAN 1029 00:52:51,200 --> 00:52:51,800 TRULY PUT SOMETHING LIKE THIS 1030 00:52:51,800 --> 00:52:56,000 IN PLACE AND DRIVE EXECUTION 1031 00:52:56,000 --> 00:53:00,200 AS A PATIENT IT DOES BRING 1032 00:53:00,200 --> 00:53:03,040 HOPE. 1033 00:53:03,040 --> 00:53:03,720 >>: THANK YOU ED. WERE 1034 00:53:03,720 --> 00:53:08,400 GOING TO ADDRESSES FOR COOPER. 1035 00:53:08,400 --> 00:53:10,240 WHAT ARE YOUR THOUGHTS AND 1036 00:53:10,240 --> 00:53:12,400 FEELINGS ON ALS RESEARCH 1037 00:53:12,400 --> 00:53:13,640 BEFORE COMPARED TO AND AFTER 1038 00:53:13,640 --> 00:53:16,160 THE DEVELOPMENT OF THE NIH/AND 1039 00:53:16,160 --> 00:53:22,560 IND AS ALS STRATEGIC PLAN. 1040 00:53:22,560 --> 00:53:23,040 >>: GOOD MORNING. 1041 00:53:23,040 --> 00:53:23,600 THINKING ABOUT THIS QUESTION 1042 00:53:23,600 --> 00:53:26,480 LAST NIGHT AND THOUGHTS REALLY 1043 00:53:26,480 --> 00:53:27,120 RETURNED TO MY MOM AS THEY SO 1044 00:53:27,120 --> 00:53:32,360 OFTEN DO. AND AFFORD A LOT OF 1045 00:53:32,360 --> 00:53:35,400 LESSONS CARING FOR HER AND ONE 1046 00:53:35,400 --> 00:53:36,480 OF WHICH WAS THE IDEA OF ALS 1047 00:53:36,480 --> 00:53:38,080 TIME WHICH IS SOMETHING I FEEL 1048 00:53:38,080 --> 00:53:38,720 LIKE I'M LIVING WITH THE NOUN 1049 00:53:38,720 --> 00:53:40,400 I THINK A LOT OF THE FOLKS WHO 1050 00:53:40,400 --> 00:53:43,080 LIVE WITH THIS DISEASE OR CARE 1051 00:53:43,080 --> 00:53:44,560 WITH FOLKS WITH THIS DISEASE 1052 00:53:44,560 --> 00:53:46,800 WILL UNDERSTAND. WE COULD 1053 00:53:46,800 --> 00:53:50,040 TALK A LOT ABOUT HOW TIME 1054 00:53:50,040 --> 00:53:50,600 EXPANDS AND CONTRACTS IN THE 1055 00:53:50,600 --> 00:53:52,880 FACE OF A HORROR LIKE ALS, BUT 1056 00:53:52,880 --> 00:53:53,440 FOR ME THE MOST SALIENT 1057 00:53:53,440 --> 00:53:55,120 DIMENSION OF TIME AND ENOUGH 1058 00:53:55,120 --> 00:53:57,480 OF IT. HORRIBLE TRAGIC THING 1059 00:53:57,480 --> 00:54:03,160 IN SOME WAYS SOME PEOPLE WHO 1060 00:54:03,160 --> 00:54:03,760 LIVE WITH DISEASES SUPERPOWERS 1061 00:54:03,760 --> 00:54:09,520 IN SOME WAYS AND WHEN YOU SEE 1062 00:54:09,520 --> 00:54:10,000 AND LIVE WITH THIS MOST 1063 00:54:10,000 --> 00:54:10,800 NIGHTMARISH DISORDER THAT FEW 1064 00:54:10,800 --> 00:54:13,680 COULD EXPERIENCE AS FAR AS I'M 1065 00:54:13,680 --> 00:54:15,120 CONCERNED, YOU HAVE A 1066 00:54:15,120 --> 00:54:17,280 DIFFERENT APPRECIATION FOR 1067 00:54:17,280 --> 00:54:17,880 SPEED AND PERHAPS A DIFFERENT 1068 00:54:17,880 --> 00:54:26,040 HUMILITY AND GRACE. AND AS I 1069 00:54:26,040 --> 00:54:28,080 GOT TO UNDERSTAND THE NIH 1070 00:54:28,080 --> 00:54:30,160 UNDERSTANDS THAT THE VALUE OF 1071 00:54:30,160 --> 00:54:31,920 THE TIME AND PEOPLE WHO LIVE 1072 00:54:31,920 --> 00:54:32,400 ON ALS TIME INTO THIS 1073 00:54:32,400 --> 00:54:36,200 IMPORTANT DECISION-MAKING 1074 00:54:36,200 --> 00:54:43,480 PROCESS IS A GOOD DEAL OF FEAR 1075 00:54:43,480 --> 00:54:49,400 AND IS A GOOD DEAL OF HOPE I 1076 00:54:49,400 --> 00:54:51,000 DO THINK WERE ON TIME .4 OF 1077 00:54:51,000 --> 00:54:53,200 THE EXPONENTIAL GROWTH CURVE 1078 00:54:53,200 --> 00:54:55,520 UP. BUT COME AWAY FEELING 1079 00:54:55,520 --> 00:54:58,440 SIMILAR BUT WITH AN 1080 00:54:58,440 --> 00:54:59,400 APPRECIATION OF EFFECT FOR 1081 00:54:59,400 --> 00:55:00,880 WHAT THIS PROCESS LOOKS LIKE 1082 00:55:00,880 --> 00:55:02,000 WHEN FOLKS LIKE US ARE 1083 00:55:02,000 --> 00:55:04,040 INCORPORATED DIRECTLY INTO THE 1084 00:55:04,040 --> 00:55:05,480 CONVERSATION. AND WHAT THEY 1085 00:55:05,480 --> 00:55:07,480 BRING TO THE TABLE AND I THINK 1086 00:55:07,480 --> 00:55:09,160 WE HAVE PRODUCED A MEANINGFUL 1087 00:55:09,160 --> 00:55:09,760 DOCUMENT AND HOW IT SERVED OUR 1088 00:55:09,760 --> 00:55:10,240 COMMUNITY WELL AND I'M 1089 00:55:10,240 --> 00:55:14,040 GRATEFUL FOR THAT. 1090 00:55:14,040 --> 00:55:16,360 >>: THANK YOU. ANDREA 1091 00:55:16,360 --> 00:55:20,800 ARE YOU OKAY WITH ME NOT 1092 00:55:20,800 --> 00:55:22,080 REREADING THE STATEMENT? 1093 00:55:22,080 --> 00:55:27,000 >>: I HAVE IT THANK YOU. 1094 00:55:27,000 --> 00:55:27,640 I WOULD SAY THAT MY EXPERIENCE 1095 00:55:27,640 --> 00:55:30,480 IN ALS RESEARCH OVER THE LAST 1096 00:55:30,480 --> 00:55:34,520 TEN-YEARS IS THAT I OBSERVED 1097 00:55:34,520 --> 00:55:34,960 THE FUNDING SYSTEM 1098 00:55:34,960 --> 00:55:38,120 PREDOMINANTLY INCENTIVIZE 1099 00:55:38,120 --> 00:55:40,080 SILOS. THAT WAS CERTAINLY IS 1100 00:55:40,080 --> 00:55:40,720 TRUE WITH BASIC SCIENCE SPACE. 1101 00:55:40,720 --> 00:55:46,640 BUT COLLABORATION THE 1102 00:55:46,640 --> 00:55:47,280 COLLABORATION OF DATA WAS MORE 1103 00:55:47,280 --> 00:55:51,280 INFREQUENT THAN EXPECTED IT TO 1104 00:55:51,280 --> 00:55:53,560 BE. AND THIS WAS DUE TO THE 1105 00:55:53,560 --> 00:55:54,880 NATURE OF THE GRANTING 1106 00:55:54,880 --> 00:55:56,440 PROCESS. I'VE SEEN SOME OF 1107 00:55:56,440 --> 00:55:58,120 THAT CHANGE AND RESEARCH IN 1108 00:55:58,120 --> 00:55:59,120 YEARS BUT IT WAS NOT CHANGING 1109 00:55:59,120 --> 00:55:59,720 QUICKLY ENOUGH TO HELP PEOPLE 1110 00:55:59,720 --> 00:56:03,760 LIVING WITH ALS. I BELIEVE 1111 00:56:03,760 --> 00:56:04,800 THE CHANGE HAS BEGUN TO TAKE 1112 00:56:04,800 --> 00:56:05,360 PLACE AS MORE OPPORTUNITIES 1113 00:56:05,360 --> 00:56:07,760 HAVE ARISEN WITH PEOPLE 1114 00:56:07,760 --> 00:56:11,400 GETTING ALS TO GET INVOLVED. 1115 00:56:11,400 --> 00:56:12,000 WHERE THEY ARE WITH THIS PLAN 1116 00:56:12,000 --> 00:56:15,520 TODAY REALLY SHOWS A WIDE 1117 00:56:15,520 --> 00:56:20,120 RANGE OF OBJECTIVES THAT WE 1118 00:56:20,120 --> 00:56:20,960 DESCRIBED DESIGNED TO 1119 00:56:20,960 --> 00:56:21,560 ACCELERATE RESEARCH TO MOVE 1120 00:56:21,560 --> 00:56:23,320 MORE QUICKLY IN TRANSLATIONAL 1121 00:56:23,320 --> 00:56:29,040 RESEARCH AND THE ESTABLISHMENT 1122 00:56:29,040 --> 00:56:30,200 OF DATA PLATFORMS AND RESEARCH 1123 00:56:30,200 --> 00:56:34,040 LANDSCAPES THAT WILL FOSTER 1124 00:56:34,040 --> 00:56:35,000 COOPERATION. I BELIEVE THAT 1125 00:56:35,000 --> 00:56:39,120 IS THE ANY WORK WE NEED TO DO 1126 00:56:39,120 --> 00:56:41,600 HERE. I BELIEVE WE NEED TO 1127 00:56:41,600 --> 00:56:42,480 OPERATIONALIZE HIS PLAN AND 1128 00:56:42,480 --> 00:56:42,720 I'M READY. 1129 00:56:42,720 --> 00:56:50,840 >>: THANK YOU. STEVE. 1130 00:56:50,840 --> 00:56:53,600 >>: YES, SO MY OPINION ON 1131 00:56:53,600 --> 00:56:55,880 RESEARCH PRIOR TO THIS 1132 00:56:55,880 --> 00:57:03,120 PLANNING EFFORT TODAY HAS 1133 00:57:03,120 --> 00:57:08,240 CHANGED SLIGHTLY. I QUICKLY 1134 00:57:08,240 --> 00:57:09,960 BECAME A RESEARCH PARTICIPANT 1135 00:57:09,960 --> 00:57:10,640 AND MANY RETRIALS ARE 1136 00:57:10,640 --> 00:57:12,800 QUALIFIED FOR. I QUICKLY 1137 00:57:12,800 --> 00:57:13,320 LEARNED THAT THE CLINICAL 1138 00:57:13,320 --> 00:57:17,440 TRIALS ARE EXTREMELY COMPLEX. 1139 00:57:17,440 --> 00:57:18,160 THE VENUE AND THE COMPLEXITY 1140 00:57:18,160 --> 00:57:19,880 OF THE DISEASE LIKE AL SS AND 1141 00:57:19,880 --> 00:57:22,520 AT TIMES FROM A PATIENT'S 1142 00:57:22,520 --> 00:57:23,040 PERSPECTIVE VACANCY RATHER 1143 00:57:23,040 --> 00:57:27,120 DAUNTING. SO, INSTEAD OF 1144 00:57:27,120 --> 00:57:30,440 BEING FEARFUL THAT I HAVE 1145 00:57:30,440 --> 00:57:31,120 LEANED IN AND BECOMING 1146 00:57:31,120 --> 00:57:31,960 INVOLVED IS HELPED EDUCATE ME 1147 00:57:31,960 --> 00:57:32,680 ALONG THE WAY IT IS BUT A FIVE 1148 00:57:32,680 --> 00:57:36,040 YEAR JOURNEY OF LEARNING 1149 00:57:36,040 --> 00:57:36,640 THROUGH THE PROCESS OF BEING 1150 00:57:36,640 --> 00:57:43,080 ENGAGED IN RESEARCH. WHAT I 1151 00:57:43,080 --> 00:57:45,520 HAVE LEARNED IS THAT RESEARCH 1152 00:57:45,520 --> 00:57:50,640 IS INFORMED AND WILL HELP 1153 00:57:50,640 --> 00:57:52,920 RESEARCH CLINICIANS AND PROFIT 1154 00:57:52,920 --> 00:57:53,680 AND NONPROFIT SPONSORS. 1155 00:57:53,680 --> 00:57:56,560 DOCTOR BROWN BENCH OF THE PIE 1156 00:57:56,560 --> 00:57:57,160 PLATE OF CURRENTLY EXISTS THAT 1157 00:57:57,160 --> 00:58:01,760 IS GETTING LARGER AND IS IN 1158 00:58:01,760 --> 00:58:02,600 NEED OF EXPERTISE AND 1159 00:58:02,600 --> 00:58:05,920 GUIDANCE. I THINK IN MANY 1160 00:58:05,920 --> 00:58:08,440 WAYS ALSO AT KELLS ON THIS 1161 00:58:08,440 --> 00:58:11,000 PANEL HAVE CONTRIBUTED TO HELP 1162 00:58:11,000 --> 00:58:11,520 HUMANIZE THAT PROCESS AND 1163 00:58:11,520 --> 00:58:16,200 FOSTER FURTHER COOPERATION AND 1164 00:58:16,200 --> 00:58:20,440 IS TRAPPED THERE GREAT 1165 00:58:20,440 --> 00:58:22,400 INVESTMENT. 1166 00:58:22,400 --> 00:58:26,480 >>: THANK YOU STEVE. 1167 00:58:26,480 --> 00:58:30,480 ALLISON. 1168 00:58:30,480 --> 00:58:32,280 >>: THANK YOU. BECAUSE 1169 00:58:32,280 --> 00:58:34,360 OF MY INVOLVEMENT IN OR WITH 1170 00:58:34,360 --> 00:58:35,120 DIFFERENT ORGANIZATIONS WITHIN 1171 00:58:35,120 --> 00:58:37,200 THE ALS COMMUNITY I TEND TO 1172 00:58:37,200 --> 00:58:39,360 HAVE A VERY HOLISTIC VIEW OF 1173 00:58:39,360 --> 00:58:40,360 THE ALS RESEARCH LANDSCAPE. 1174 00:58:40,360 --> 00:58:43,000 WHEN I SIT DOWN OF THE VIRTUAL 1175 00:58:43,000 --> 00:58:44,200 TABLE WITH NUMBERS OF ALL OF 1176 00:58:44,200 --> 00:58:45,040 THESE LARGE ORGANIZATIONS 1177 00:58:45,040 --> 00:58:46,400 WITHIN THE COMMUNITY I WASN'T 1178 00:58:46,400 --> 00:58:48,560 REALLY SURPRISED TO HEAR THAT 1179 00:58:48,560 --> 00:58:50,600 WE ACTUALLY HAVE QUITE A BIT 1180 00:58:50,600 --> 00:58:51,960 IN COMMON. WE TALKED ABOUT 1181 00:58:51,960 --> 00:58:53,680 OUR PRIORITIES AND OUR GOALS 1182 00:58:53,680 --> 00:58:55,280 AND THERE IS TOO MUCH ERROR IN 1183 00:58:55,280 --> 00:58:56,800 A STRONG COLLABORATION AMONGST 1184 00:58:56,800 --> 00:58:58,760 US THAT REALLY REINFORCE THE 1185 00:58:58,760 --> 00:59:00,080 DESIRE TO CREATE MEANINGFUL 1186 00:59:00,080 --> 00:59:01,920 SOLUTIONS TO FINDING REAL 1187 00:59:01,920 --> 00:59:04,200 ANSWERS AND TO NOT RE-CREATING 1188 00:59:04,200 --> 00:59:05,000 OR DUPLICATING EFFORTS. WE 1189 00:59:05,000 --> 00:59:07,120 TRULY CAME IN FOR SESSIONS 1190 00:59:07,120 --> 00:59:12,320 WITH AN ABUNDANCE OF HOWEVER, 1191 00:59:12,320 --> 00:59:14,440 I THINK I SHARE ON THIS ONE MY 1192 00:59:14,440 --> 00:59:16,000 CONCERN COMING OUT OF THIS IS 1193 00:59:16,000 --> 00:59:17,600 THAT AN ABUNDANCE OF IDEAS 1194 00:59:17,600 --> 00:59:19,040 REQUIRES AN ABUNDANCE OF 1195 00:59:19,040 --> 00:59:21,040 FUNDING AND RESOURCES TO TURN 1196 00:59:21,040 --> 00:59:22,760 INTO REALITY. I'M TRULY NOT 1197 00:59:22,760 --> 00:59:23,480 SURE WITH THE FUNDING WILL 1198 00:59:23,480 --> 00:59:27,080 COME FROM OR HOW PEOPLE GO 1199 00:59:27,080 --> 00:59:31,080 ABOUT MAKING GOOD JOB OF 1200 00:59:31,080 --> 00:59:31,520 WORKING TOGETHER AND 1201 00:59:31,520 --> 00:59:32,080 ARTICULATE OUR DIVISIONS AND 1202 00:59:32,080 --> 00:59:34,760 WE KNOW THAT REALISTICALLY WE 1203 00:59:34,760 --> 00:59:35,400 NEED TO ACKNOWLEDGE THAT HER 1204 00:59:35,400 --> 00:59:36,800 VISION ALONE WILL NOT GET US 1205 00:59:36,800 --> 00:59:39,120 TO WHERE WE NEED TO BE SAID 1206 00:59:39,120 --> 00:59:40,320 THAT PIECE OF TURNING GREAT 1207 00:59:40,320 --> 00:59:42,200 IDEAS INTO GREAT REAL CHANGES 1208 00:59:42,200 --> 00:59:44,000 IS STILL A LITTLE UNCERTAIN 1209 00:59:44,000 --> 00:59:46,120 FOR ME. I'M HOPEFUL, ANXIOUS, 1210 00:59:46,120 --> 00:59:46,760 AND LOOKING FORWARD TO SEEING 1211 00:59:46,760 --> 00:59:51,680 WHERE THE STRATEGIC PLAN GOES. 1212 00:59:51,680 --> 00:59:52,400 >>: THANK YOU ALLISON. I 1213 00:59:52,400 --> 00:59:53,000 DON'T BELIEVE THAT NADIA HAS 1214 00:59:53,000 --> 01:00:02,400 ARRIVED YET. THERE YOU ARE. 1215 01:00:02,400 --> 01:00:11,200 OKAY, NADIA. HAVE YOU SEEN 1216 01:00:11,200 --> 01:00:14,960 THE PEOPLE ARE RESPONDING TO? 1217 01:00:14,960 --> 01:00:16,840 AND ALSO INTRODUCE YOURSELF 1218 01:00:16,840 --> 01:00:17,080 PLEASE. 1219 01:00:17,080 --> 01:00:17,840 >>: HI EVERYBODY, I AM 1220 01:00:17,840 --> 01:00:19,560 NADIA AND IT IS MY PLEASURE TO 1221 01:00:19,560 --> 01:00:20,440 BE HERE. I APOLOGIZE, I HAD 1222 01:00:20,440 --> 01:00:24,840 TO DROP THE KIDS OFF AT 1223 01:00:24,840 --> 01:00:25,520 SCHOOL. LIFE GOES ON AS WE 1224 01:00:25,520 --> 01:00:29,600 TRY TO FIGHT ALS. IT'S MY 1225 01:00:29,600 --> 01:00:31,240 PLEASURE TO BE HERE AND I'VE 1226 01:00:31,240 --> 01:00:32,840 BEEN SO INVOLVED IN ADVOCACY 1227 01:00:32,840 --> 01:00:35,040 THE LAST FEW YEARS I LOST MY 1228 01:00:35,040 --> 01:00:36,720 HUSBAND TO A FAST, PROGRESSIVE 1229 01:00:36,720 --> 01:00:39,960 FORM OF ALS IN 2020. TO MY 1230 01:00:39,960 --> 01:00:40,760 SURPRISE, THERE WAS A GENETIC 1231 01:00:40,760 --> 01:00:42,960 COMPONENT TO IT ALTHOUGH WE 1232 01:00:42,960 --> 01:00:44,520 WERE TO SHARE A FAMILY HISTORY 1233 01:00:44,520 --> 01:00:46,640 AT THAT POINT. I AM GRATEFUL 1234 01:00:46,640 --> 01:00:48,280 FOR THE NIH EFFORTS AND I 1235 01:00:48,280 --> 01:00:51,160 THINK THAT MY UNDERSTANDING OF 1236 01:00:51,160 --> 01:00:52,640 RESEARCH BEFORE THIS MEETING 1237 01:00:52,640 --> 01:00:58,400 I'VE LEARNED A LOT. SPENT 1238 01:00:58,400 --> 01:00:59,680 FAST LEARNING IN THE PAST 1239 01:00:59,680 --> 01:01:01,120 COUPLE OF MONTHS WITH PEOPLE 1240 01:01:01,120 --> 01:01:03,040 WHO ARE ARE WORKING ON A 1241 01:01:03,040 --> 01:01:04,160 CONCERTED EFFORT TO CHANGE. I 1242 01:01:04,160 --> 01:01:08,320 THINK BEFORE I HAD THIS GROUP 1243 01:01:08,320 --> 01:01:09,840 AND I THINK THEY'RE EVEN MORE 1244 01:01:09,840 --> 01:01:11,480 COMPLICATED BUT NOW I'M GLAD 1245 01:01:11,480 --> 01:01:12,040 THERE'S A CONCERTED EFFORT 1246 01:01:12,040 --> 01:01:13,200 FROM MANY DIFFERENT PARTIES. 1247 01:01:13,200 --> 01:01:17,880 I DO THINK THAT YOU TAKE. WE 1248 01:01:17,880 --> 01:01:19,920 NEED THE RESEARCH AND ACADEMIC 1249 01:01:19,920 --> 01:01:23,880 PERSPECTIVE AND MANY OF THE 1250 01:01:23,880 --> 01:01:24,640 VOICE OF PATIENTS AND 1251 01:01:24,640 --> 01:01:25,360 CAREGIVERS AS WELL. THIS WAS 1252 01:01:25,360 --> 01:01:29,080 A MOST EXCITING ABOUT. I 1253 01:01:29,080 --> 01:01:31,440 THINK BE ON MY OWN PERSONAL 1254 01:01:31,440 --> 01:01:32,600 PERSPECTIVE IT'S GOING TO 1255 01:01:32,600 --> 01:01:33,560 MATTER IS HOW THE COMMUNITY 1256 01:01:33,560 --> 01:01:35,240 RESPONSE TO WHAT WE SHARE IN 1257 01:01:35,240 --> 01:01:35,840 THE NEXT TWO-DAYS AND THE 1258 01:01:35,840 --> 01:01:39,480 STEPS THAT COME OUT OF THAT. 1259 01:01:39,480 --> 01:01:41,320 THOSE ARE THE THINGS ARE GOING 1260 01:01:41,320 --> 01:01:42,760 TO BE IMPACTFUL. I AM HOPING 1261 01:01:42,760 --> 01:01:43,640 THAT ANOTHER PRODUCT FROM THE 1262 01:01:43,640 --> 01:01:49,720 NEXT TWO-DAYS WILL BE THAT THE 1263 01:01:49,720 --> 01:01:50,200 PATIENT AND CAREGIVER 1264 01:01:50,200 --> 01:01:51,920 COMMUNITY WILL CONTINUE TO 1265 01:01:51,920 --> 01:01:53,880 ADVOCATE FOR A VERY STRONG 1266 01:01:53,880 --> 01:01:55,640 SCIENCE. THAT'S WHAT WE NEED 1267 01:01:55,640 --> 01:01:56,320 TO REALLY MAKE A DIFFERENCE IN 1268 01:01:56,320 --> 01:02:03,000 ALS AND IN THE FUTURE. THERE 1269 01:02:03,000 --> 01:02:04,800 ARE THINGS ALREADY IN PLACE 1270 01:02:04,800 --> 01:02:06,200 THAT ARE MOVING FORWARD AND 1271 01:02:06,200 --> 01:02:07,640 THERE'S MANY CLINICAL TILES 1272 01:02:07,640 --> 01:02:08,360 TRIALS AND GREAT RESEARCH IS 1273 01:02:08,360 --> 01:02:10,960 HAPPENING. WHAT WERE GOING TO 1274 01:02:10,960 --> 01:02:12,240 NEED MORE FOR ALS AND WE HAVE 1275 01:02:12,240 --> 01:02:13,560 TO CONTINUE TO ADVOCATE FOR 1276 01:02:13,560 --> 01:02:14,120 STRONG SCIENCE AND CONCERTED 1277 01:02:14,120 --> 01:02:15,640 EFFORTS TO MAKE THAT HAPPEN. 1278 01:02:15,640 --> 01:02:22,200 >>: THANK YOU. 1279 01:02:22,200 --> 01:02:24,320 >>: GO AHEAD ELLEN YOU 1280 01:02:24,320 --> 01:02:24,840 CAN ANSWER THE QUESTION 1281 01:02:24,840 --> 01:02:28,880 YOURSELF. YES AND THEN YOU 1282 01:02:28,880 --> 01:02:29,840 CAN. 1283 01:02:29,840 --> 01:02:32,880 >>: IT'S HARD TO BE BRIEF 1284 01:02:32,880 --> 01:02:38,560 WHEN MY HUSBAND DIED IN 1984. 1285 01:02:38,560 --> 01:02:39,880 SO, I HAVE SEEN A LOT BEFORE 1286 01:02:39,880 --> 01:02:40,400 OBVIOUSLY NOT GOING TO GO 1287 01:02:40,400 --> 01:02:45,840 THROUGH EVERY YEAR OR EVEN THE 1288 01:02:45,840 --> 01:02:47,920 AID BUT THERE'S A COUPLE OF 1289 01:02:47,920 --> 01:02:50,040 THINGS THAT STAND OUT AND ONE 1290 01:02:50,040 --> 01:02:52,400 IS IN THE LATE 1980s THE ALS 1291 01:02:52,400 --> 01:02:54,080 ASSOCIATION HAVE APPLICANT CJ 1292 01:02:54,080 --> 01:02:59,840 AND A BUNCH OF US WENT TO NIH, 1293 01:02:59,840 --> 01:03:00,840 KNOCKED ON THE DOOR AND SAID 1294 01:03:00,840 --> 01:03:04,880 HE WAS THE PERSON IN CHARGE OF 1295 01:03:04,880 --> 01:03:06,440 THE ALS RESEARCH HERE? AND 1296 01:03:06,440 --> 01:03:07,040 AFTER A TIME A GENTLEMAN CAME 1297 01:03:07,040 --> 01:03:10,640 OUT AND HE SAID, WHAT ARE YOU 1298 01:03:10,640 --> 01:03:12,600 DOING FOR ALS? HE SAID 1299 01:03:12,600 --> 01:03:18,040 NOTHING, NO ONE IS INTERESTED. 1300 01:03:18,040 --> 01:03:18,640 HE COULD'VE HAD A LITTLE MORE 1301 01:03:18,640 --> 01:03:20,800 POLISH TO THE ANSWER BUT WHAT 1302 01:03:20,800 --> 01:03:23,800 I FOUND THAT HE MEANT WAS THAT 1303 01:03:23,800 --> 01:03:27,760 NO ONE WAS APPLYING FOR 1304 01:03:27,760 --> 01:03:28,320 GRANTS. AND THEREFORE HE 1305 01:03:28,320 --> 01:03:32,360 DEDUCED THAT NO ONE IS 1306 01:03:32,360 --> 01:03:34,720 INTERESTED. THROUGHOUT THIS 1307 01:03:34,720 --> 01:03:35,240 TIME I REMEMBER SAYING TO 1308 01:03:35,240 --> 01:03:39,400 MYSELF WHEN MY DAUGHTER WAS 1309 01:03:39,400 --> 01:03:44,520 THEN AS I SAID, SHE WAS THREE 1310 01:03:44,520 --> 01:03:45,160 WHEN MY HUSBAND DIED. WHEN HE 1311 01:03:45,160 --> 01:03:51,680 WOULD GET OLDER I KEPT HOPING 1312 01:03:51,680 --> 01:03:52,440 AND A FEW MORE YEARS. THERE 1313 01:03:52,440 --> 01:03:57,480 WOULD BE SOME TYPE OF DRUG OR 1314 01:03:57,480 --> 01:04:01,480 CURE. I REALIZED EVERY FEW 1315 01:04:01,480 --> 01:04:04,520 YEARS I MOVED UP WITH THE HOPE 1316 01:04:04,520 --> 01:04:06,880 THAT WHEN SHE IS TEN, WHEN SHE 1317 01:04:06,880 --> 01:04:14,360 IS 16 AND TIME HAD FLOWN BY 1318 01:04:14,360 --> 01:04:16,640 AND THERE STILL NOT HOPE THAT 1319 01:04:16,640 --> 01:04:18,920 WE NEED AND THE CONCRETE 1320 01:04:18,920 --> 01:04:24,280 DRUGS. BUT HAVING SERVED ON 1321 01:04:24,280 --> 01:04:25,640 THIS I CAN SEE THE LANDSCAPE 1322 01:04:25,640 --> 01:04:29,680 CHANGE. I'M SO IMPRESSED WITH 1323 01:04:29,680 --> 01:04:31,000 EVERYONE WHO HAVE PARTICIPATED 1324 01:04:31,000 --> 01:04:33,920 WITH THE AMOUNT OF DETAIL IN 1325 01:04:33,920 --> 01:04:39,120 THE AMOUNT OF GRIT ALL OF THE 1326 01:04:39,120 --> 01:04:46,560 THINKING THIS THROUGH AND VERY 1327 01:04:46,560 --> 01:04:49,720 HOPEFUL. AND ONLY TYPE TIME 1328 01:04:49,720 --> 01:04:52,240 WILL TELL. 1329 01:04:52,240 --> 01:05:00,040 >>: THANK YOU. THIS IS 1330 01:05:00,040 --> 01:05:02,880 IMPRESSIVE AND MAJOR GROUP OF 1331 01:05:02,880 --> 01:05:03,680 PEOPLE. NOT JUST THE ALS 1332 01:05:03,680 --> 01:05:04,160 EXPERIENCE EXPERTS, BUT 1333 01:05:04,160 --> 01:05:08,280 EVERYBODY INVOLVED. I HAVE 1334 01:05:08,280 --> 01:05:10,480 SEEN TIME AND AGAIN PEOPLE 1335 01:05:10,480 --> 01:05:12,640 CARING ABOUT WHAT WE HAVE TO 1336 01:05:12,640 --> 01:05:23,120 SAY. THAT IS SO IMPORTANT. 1337 01:05:26,040 --> 01:05:26,440 MY THOUGHTS HAVE BEEN 1338 01:05:26,440 --> 01:05:28,160 MENTIONED AND TEARING DOWN 1339 01:05:28,160 --> 01:05:34,280 SILOS AND REDUNDANCIES IS SO 1340 01:05:34,280 --> 01:05:41,160 IMPORTANT THIS PLAN WHICH I 1341 01:05:41,160 --> 01:05:44,400 CALL A ROADMAP I HOPE THIS 1342 01:05:44,400 --> 01:05:45,720 ROADMAP WILL RESULT IN A 1343 01:05:45,720 --> 01:05:52,800 SYNERGISTIC OPPORTUNITY. I 1344 01:05:52,800 --> 01:05:55,000 REALLY HOPE THAT I'M 1345 01:05:55,000 --> 01:05:56,000 OPTIMISTIC. EVEN THOUGH I 1346 01:05:56,000 --> 01:06:00,000 HAVE ALS I'M STILL AN 1347 01:06:00,000 --> 01:06:02,160 OPTIMIST. AND I THINK THIS 1348 01:06:02,160 --> 01:06:02,760 PLAN WILL LEND ITSELF TO MORE 1349 01:06:02,760 --> 01:06:07,400 SYNERGISTIC OPPORTUNITIES. 1350 01:06:07,400 --> 01:06:10,160 WITH THAT THANK YOU. GOING TO 1351 01:06:10,160 --> 01:06:13,000 HAVE ANOTHER SESSION TOMORROW 1352 01:06:13,000 --> 01:06:15,120 MORNING WITH THE SAME AMAZING 1353 01:06:15,120 --> 01:06:20,880 GROUP OF PEOPLE. AND WITH 1354 01:06:20,880 --> 01:06:22,680 THAT LIKE TO TURN THE MEETING 1355 01:06:22,680 --> 01:06:27,640 OVER TO THE COCHAIRS WHO ARE 1356 01:06:27,640 --> 01:06:28,960 DOING EXCEL UNIT RESEARCH ON 1357 01:06:28,960 --> 01:06:29,560 THE BIOLOGY BEHIND ALS FOR OUR 1358 01:06:29,560 --> 01:06:31,640 NEXT ACTION. CAN WE TURN THE 1359 01:06:31,640 --> 01:06:41,640 SLIDE PLEASE? THANK YOU. 1360 01:06:41,640 --> 01:06:45,920 >>: THANK YOU EVERYONE ON 1361 01:06:45,920 --> 01:06:47,280 THE LIVED EXPERIENCE PANEL. 1362 01:06:47,280 --> 01:06:49,400 WE WANT TO REMIND WORKSHOP 1363 01:06:49,400 --> 01:06:50,040 ATTENDEES THAT THEY MAY ENTER 1364 01:06:50,040 --> 01:06:50,600 QUESTIONS INTO THE ZOOM 1365 01:06:50,600 --> 01:06:52,320 QUESTION AND ANSWER BOX. OR 1366 01:06:52,320 --> 01:06:56,000 THEY CAN FILL OUT THE QUESTION 1367 01:06:56,000 --> 01:06:57,400 AND ANSWER FORM ON THE 1368 01:06:57,400 --> 01:06:59,360 WEBSITE. GOING TO BE TALKING 1369 01:06:59,360 --> 01:07:01,280 ABOUT THE ACCELERATING 1370 01:07:01,280 --> 01:07:01,920 RESEARCH ON THE BIOLOGY BEHIND 1371 01:07:01,920 --> 01:07:02,800 ALS WORKING GROUP. CAN I HAVE 1372 01:07:02,800 --> 01:07:11,600 THE NEXT SLIDE PLEASE. I AM 1373 01:07:11,600 --> 01:07:13,640 WORKING AT KING'S COLLEGE 1374 01:07:13,640 --> 01:07:15,280 LONDON AS A NEUROLOGIST. MY 1375 01:07:15,280 --> 01:07:17,360 COCHAIR IS FROM HARVARD 1376 01:07:17,360 --> 01:07:21,240 MEDICAL SCHOOL AT 1377 01:07:21,240 --> 01:07:21,720 MASSACHUSETTS GENERAL 1378 01:07:21,720 --> 01:07:22,680 HOSPITAL. WE HAVE MANY 1379 01:07:22,680 --> 01:07:24,600 MEMBERS IN OUR WORKING GROUP 1380 01:07:24,600 --> 01:07:25,880 WILL BE INTRODUCING THEM IN 1381 01:07:25,880 --> 01:07:26,440 MORE DETAIL AFTER THE 1382 01:07:26,440 --> 01:07:28,360 PRESENTATION. BUT TO QUICKLY 1383 01:07:28,360 --> 01:07:38,800 NAME THEM TO HAVE THE NEXT 1384 01:07:43,600 --> 01:07:51,800 SLIDE PLEASE. SO, OUR THREE 1385 01:07:51,800 --> 01:07:54,760 PRIORITIES OF YOU ALREADY 1386 01:07:54,760 --> 01:07:55,520 HEARD ARE: TO UNLOCK SPORADIC 1387 01:07:55,520 --> 01:07:55,960 ALS TO IDENTIFY NEW 1388 01:07:55,960 --> 01:07:59,720 THERAPEUTIC TARGETS ACROSS 1389 01:07:59,720 --> 01:08:01,800 ANCESTRIES. AND PRIORITY TWO: 1390 01:08:01,800 --> 01:08:04,360 IS TO UNDERSTAND THE MOLECULAR 1391 01:08:04,360 --> 01:08:04,960 MECHANISMS UNDERLYING CLINICAL 1392 01:08:04,960 --> 01:08:06,240 HETEROGENEITY IN ALS. THE 1393 01:08:06,240 --> 01:08:08,360 THIRD PRIORITY: IS TO HARNESS 1394 01:08:08,360 --> 01:08:08,880 EMERGING TECHNOLOGIES TO 1395 01:08:08,880 --> 01:08:09,480 UNCOVER NEW DISEASE MECHANISMS 1396 01:08:09,480 --> 01:08:13,600 AND MECHANISMS OF ALS. I WILL 1397 01:08:13,600 --> 01:08:15,040 GO THROUGH EACH OF THOSE AND 1398 01:08:15,040 --> 01:08:16,680 EXPLAIN IN MORE DETAIL WHAT WE 1399 01:08:16,680 --> 01:08:18,840 MEAN. IF YOU HAVE THE NEXT 1400 01:08:18,840 --> 01:08:20,800 SLIDE PLEASE. THE FIRST 1401 01:08:20,800 --> 01:08:21,360 PRIORITY TO UNLOCK SPORADIC 1402 01:08:21,360 --> 01:08:21,920 ALS. IF YOU HAVE A LOOK AT 1403 01:08:21,920 --> 01:08:25,440 THE DIAGRAM ON THE RIGHT: 1404 01:08:25,440 --> 01:08:28,360 THIS IS A PIE CHART SHOWING 1405 01:08:28,360 --> 01:08:29,840 THE CONTRIBUTIONS OF FAMILIAL 1406 01:08:29,840 --> 01:08:32,920 ALS AND SPORADIC ALS AND 1407 01:08:32,920 --> 01:08:34,360 GENETIC NONGENETIC FORMS OF 1408 01:08:34,360 --> 01:08:35,640 ALS. THE BLUE SHADING IS 1409 01:08:35,640 --> 01:08:37,280 PEOPLE WOULD SPORADIC ALS, 1410 01:08:37,280 --> 01:08:39,480 PEOPLE WITH NO OBVIOUS FAMILY 1411 01:08:39,480 --> 01:08:41,040 HISTORY. AND THE RED IS 1412 01:08:41,040 --> 01:08:41,920 PEOPLE WITH FAMILIAL ALS, 1413 01:08:41,920 --> 01:08:44,520 PEOPLE WITH A FAMILY HISTORY 1414 01:08:44,520 --> 01:08:46,240 OF THE DISEASE. AND YOU CAN 1415 01:08:46,240 --> 01:08:47,200 SEE ALONG THE TOP THAT I HAVE 1416 01:08:47,200 --> 01:08:48,440 BRACKETED THE TWO LARGE 1417 01:08:48,440 --> 01:08:52,720 SEGMENTS IN BLUE AND RED. 1418 01:08:52,720 --> 01:08:54,280 THOSE ARE PEOPLE WITH SPORADIC 1419 01:08:54,280 --> 01:08:55,960 ALS AND FAMILIAL ALS FOR THAT 1420 01:08:55,960 --> 01:08:57,680 IS UNKNOWN AND JANE. TO BE OF 1421 01:08:57,680 --> 01:08:58,880 SOME UNDERSTANDING OF WHAT'S 1422 01:08:58,880 --> 01:09:03,080 CAUSING IT AND THOSE PEOPLE. 1423 01:09:03,080 --> 01:09:04,720 C. THEN IF YOU CAN SEE THE 1424 01:09:04,720 --> 01:09:05,520 SMALL SLIVER OF RED ON THE 1425 01:09:05,520 --> 01:09:09,640 RIGHT AND THEN THE MUCH LARGER 1426 01:09:09,640 --> 01:09:12,320 BLUE SPORADIC ALS SECTION, 1427 01:09:12,320 --> 01:09:13,600 THAT IS PEOPLE WHO HAVE NO 1428 01:09:13,600 --> 01:09:15,200 KNOWN GENE. WE HAVE VERY 1429 01:09:15,200 --> 01:09:15,960 LIMITED UNDERSTANDING OF WHY 1430 01:09:15,960 --> 01:09:19,000 THOSE PEOPLE HAVE DEVELOPED 1431 01:09:19,000 --> 01:09:21,680 ALS. WHAT WE WANT TO DO IS 1432 01:09:21,680 --> 01:09:23,080 UNDERSTAND HOW TO UNLOCK THIS 1433 01:09:23,080 --> 01:09:25,320 VERY LARGE SEGMENTS WHERE WE 1434 01:09:25,320 --> 01:09:26,240 HAVE VERY LIMITED 1435 01:09:26,240 --> 01:09:27,680 UNDERSTANDING OF THE CAUSE OF 1436 01:09:27,680 --> 01:09:30,160 ALS. ONE WAY TO DO THAT IS TO 1437 01:09:30,160 --> 01:09:31,360 ENCOURAGE CLOSE COLLABORATION 1438 01:09:31,360 --> 01:09:32,680 BETWEEN SCIENTISTS IN THE LAB 1439 01:09:32,680 --> 01:09:35,440 AND CLINICIANS TO UNDERSTAND 1440 01:09:35,440 --> 01:09:37,040 HOW TO SUBGROUP ALS. BECAUSE 1441 01:09:37,040 --> 01:09:39,600 ALS IS NOT ONE DISEASE AND WE 1442 01:09:39,600 --> 01:09:41,960 KNOW THAT VERY CLEARLY. IT'S 1443 01:09:41,960 --> 01:09:43,120 ACTUALLY LOTS OF DIFFERENT 1444 01:09:43,120 --> 01:09:43,720 CONDITIONS, BUT UNDERSTANDING 1445 01:09:43,720 --> 01:09:46,600 HOW TO BREAK IT DOWN AND HOW 1446 01:09:46,600 --> 01:09:47,120 TO CHOP UP THE DIFFERENT 1447 01:09:47,120 --> 01:09:50,200 GROUPS OF ALS AND SAY THIS IS 1448 01:09:50,200 --> 01:09:51,400 ONE GROUP AND ANOTHER GROUP 1449 01:09:51,400 --> 01:09:53,160 AND TO UNDERSTAND HOW TO 1450 01:09:53,160 --> 01:09:54,560 UNDERSTAND THE CAUSES IN THOSE 1451 01:09:54,560 --> 01:09:56,480 GROUPS IS ACTUALLY VERY 1452 01:09:56,480 --> 01:09:57,920 CHALLENGING TASKS AND WE HOPE 1453 01:09:57,920 --> 01:10:00,040 THAT THE COLLABORATION WITH 1454 01:10:00,040 --> 01:10:00,840 CLINICIANS AND TOP SCIENTIST 1455 01:10:00,840 --> 01:10:02,040 TO ENCOURAGE THAT. WE CAN GET 1456 01:10:02,040 --> 01:10:03,080 DIFFERENT VIEWS OF HOW THE 1457 01:10:03,080 --> 01:10:04,160 CLINICAL PATTERNS MATCH UP 1458 01:10:04,160 --> 01:10:07,080 WITH GOING ON IN THE 1459 01:10:07,080 --> 01:10:09,240 LABORATORY. WE ALSO NEED TO 1460 01:10:09,240 --> 01:10:10,720 DEVELOP WAYS AND SHARE VIEWS 1461 01:10:10,720 --> 01:10:12,440 WITH DIFFERENT TYPES OF DATA 1462 01:10:12,440 --> 01:10:14,120 THAT DESCRIBES ALS. OBVIOUSLY 1463 01:10:14,120 --> 01:10:15,160 THERE'S CLINICAL PATTERNS OF 1464 01:10:15,160 --> 01:10:17,360 ALS THAT WE KNOW. FOR EXAMPLE 1465 01:10:17,360 --> 01:10:18,760 WHERE IT BEGINS AND TELL THAT 1466 01:10:18,760 --> 01:10:20,800 FAST PROGRESSES AND WHETHER IT 1467 01:10:20,800 --> 01:10:23,960 IS INVOLVED THINKING AS WELL, 1468 01:10:23,960 --> 01:10:26,080 ET CETERA. THERE'S LOTS OF 1469 01:10:26,080 --> 01:10:27,240 DIFFERENT WAYS TO DESCRIBE IT 1470 01:10:27,240 --> 01:10:28,640 CLINICALLY. BUT IS ALSO OTHER 1471 01:10:28,640 --> 01:10:30,440 TYPES OF LABORATORY DATA THAT 1472 01:10:30,440 --> 01:10:33,040 WE CAN USE TO DESCRIBE ALS, 1473 01:10:33,040 --> 01:10:35,680 SUCH AS GEN X, TRANSCRIPT 1474 01:10:35,680 --> 01:10:36,640 METRICS AND ENVIRONMENTAL RISK 1475 01:10:36,640 --> 01:10:38,880 FACTORS. AND WE HAVE TO NOTE 1476 01:10:38,880 --> 01:10:40,320 THAT WHEN ONE RESEARCH GROUP 1477 01:10:40,320 --> 01:10:45,280 USES CERTAIN TERMS OR USES 1478 01:10:45,280 --> 01:10:46,120 CERTAIN DATATYPES THOSE ARE 1479 01:10:46,120 --> 01:10:48,360 UNDERSTANDABLE BY END OF A 1480 01:10:48,360 --> 01:10:49,440 GROUP. FOR EXAMPLE, WHEN 1481 01:10:49,440 --> 01:10:50,840 SOMEONE SAYS THIS IS THE AGE 1482 01:10:50,840 --> 01:10:54,200 OF ONSET FOR ALS, WHAT DO THEY 1483 01:10:54,200 --> 01:10:56,160 MEAN? THEY MEAN THE SITE OF 1484 01:10:56,160 --> 01:10:57,840 FIRST WEAKNESS? DID ANYONE 1485 01:10:57,840 --> 01:10:59,080 FORKED FLICKERING FIRST 1486 01:10:59,080 --> 01:10:59,800 HAPPENED? WHAT IS THE 1487 01:10:59,800 --> 01:11:01,240 DEFINITION? WE HAVE TO USE 1488 01:11:01,240 --> 01:11:01,680 SOME KIND OF COMMON 1489 01:11:01,680 --> 01:11:06,240 TERMINOLOGY TO ALLOW SHARING. 1490 01:11:06,240 --> 01:11:08,640 WE ALSO HAVE TO WIDEN OUR 1491 01:11:08,640 --> 01:11:09,640 UNDERSTANDING TO USE THESE 1492 01:11:09,640 --> 01:11:11,480 DIFFERENT WEE MIXES ET CETERA. 1493 01:11:11,480 --> 01:11:12,160 THE STATES TO INCLUDE 1494 01:11:12,160 --> 01:11:12,760 UNDERSTUDIED GROUPS AND THOSE 1495 01:11:12,760 --> 01:11:15,680 ARE GROUPS THAT ARE BASICALLY 1496 01:11:15,680 --> 01:11:16,880 ALL OF OUR RESEARCH AT THE 1497 01:11:16,880 --> 01:11:18,960 MOMENT HAS BEEN DONE AND 1498 01:11:18,960 --> 01:11:20,480 PEOPLE OF NORTH EUROPEAN 1499 01:11:20,480 --> 01:11:21,120 ANCESTRY. WE NEED TO INCLUDE 1500 01:11:21,120 --> 01:11:23,040 EVERYBODY BECAUSE HE 1501 01:11:23,040 --> 01:11:25,320 UNDERSTUDIED GROUPS HAVE MANY 1502 01:11:25,320 --> 01:11:27,040 OF THE BIOLOGICAL ANSWERS THAT 1503 01:11:27,040 --> 01:11:28,800 WE NEED AND MANY OF THE 1504 01:11:28,800 --> 01:11:29,640 ENVIRONMENTAL ANSWERS THAT WE 1505 01:11:29,640 --> 01:11:32,800 NEED TO UNLOCK SPORADIC ALS. 1506 01:11:32,800 --> 01:11:34,400 THE OTHER REASON WE NEED TO 1507 01:11:34,400 --> 01:11:35,360 INCLUDE UNDERSTANDING THESE 1508 01:11:35,360 --> 01:11:36,600 GROUPS IS BECAUSE TREATMENT IS 1509 01:11:36,600 --> 01:11:38,080 APPLIED TO PEOPLE OF MORE 1510 01:11:38,080 --> 01:11:39,240 EUROPEAN ANCESTRY MAY NOT 1511 01:11:39,240 --> 01:11:40,800 APPLY TO PEOPLE OF OTHER 1512 01:11:40,800 --> 01:11:41,480 ANCESTRIES. AND SO, THERE'S A 1513 01:11:41,480 --> 01:11:46,640 VERY IMPORTANT REASON TO 1514 01:11:46,640 --> 01:11:48,400 GENEALOGY. AND THE OTHER 1515 01:11:48,400 --> 01:11:49,720 ASPECT THAT IS VERY IMPORTANT 1516 01:11:49,720 --> 01:11:51,520 TO UNLOCK HIS PRODUCT ALS IS 1517 01:11:51,520 --> 01:11:52,720 TO DEVELOP NEW WAYS TO MODEL 1518 01:11:52,720 --> 01:11:54,560 ALS IN THE LAB AND MAKE 1519 01:11:54,560 --> 01:11:56,600 INTRODUCE REFLECTIVE AND 1520 01:11:56,600 --> 01:11:57,600 PATHOLOGY. AT THE MOMENT 1521 01:11:57,600 --> 01:11:58,240 NEARLY ALL MODELS INVOLVE 1522 01:11:58,240 --> 01:11:59,720 USING GENES THAT ARE BEING 1523 01:11:59,720 --> 01:12:01,680 CHANGED IN SOME WAY. SO, SOME 1524 01:12:01,680 --> 01:12:05,720 GENE MUTATIONS IMPORTANT FOR 1525 01:12:05,720 --> 01:12:09,640 ALS PEOPLE WITH THIS GENETIC 1526 01:12:09,640 --> 01:12:10,880 BASIS FOR ALS HAD REMODELED 1527 01:12:10,880 --> 01:12:14,960 THE LAB CORRECTLY. THAT IS A 1528 01:12:14,960 --> 01:12:15,720 CHALLENGING ISSUE BUT IT'S 1529 01:12:15,720 --> 01:12:16,480 IMPORTANT WHEN WE MUST 1530 01:12:16,480 --> 01:12:20,600 RESOLVE. THE NEXT SLIDE 1531 01:12:20,600 --> 01:12:23,960 PLEASE. THE SECOND PRIORITY 1532 01:12:23,960 --> 01:12:24,560 IS TO UNDERSTAND THE MOLECULAR 1533 01:12:24,560 --> 01:12:28,240 MECHANISMS UNDERSTANDING THE 1534 01:12:28,240 --> 01:12:29,840 CLINICAL HETEROGENEITY IS THE 1535 01:12:29,840 --> 01:12:31,080 DIFFERENCES IN THE WAY TO ALS 1536 01:12:31,080 --> 01:12:32,640 PRESENTS. ADVANTAGES AND AGE 1537 01:12:32,640 --> 01:12:34,480 OF ONSET, CLINICAL FEATURES 1538 01:12:34,480 --> 01:12:35,200 AND PROGRESSION RATES. AGAIN 1539 01:12:35,200 --> 01:12:36,920 IF YOU LOOK AT THE DIAGRAM ON 1540 01:12:36,920 --> 01:12:39,200 THE RIGHTS, YOU CAN SEE THERE 1541 01:12:39,200 --> 01:12:42,840 IS A DNA WITH A DOUBLE HELIX 1542 01:12:42,840 --> 01:12:44,640 IN THE RED STAR INDICATING A 1543 01:12:44,640 --> 01:12:45,680 MUTATION. AND THERE IS A 1544 01:12:45,680 --> 01:12:48,280 PERSON WHO HAS SOME RISK 1545 01:12:48,280 --> 01:12:49,360 FACTORS THAT WILL PUSH THEM 1546 01:12:49,360 --> 01:12:50,040 TOWARDS DEVELOPING ALS OR 1547 01:12:50,040 --> 01:12:53,880 FRONTOTEMPORAL DEMENTIA OR 1548 01:12:53,880 --> 01:12:54,680 BOTH. THERE'S ALSO SOME 1549 01:12:54,680 --> 01:12:55,160 RESILIENCE FACTORS THAT 1550 01:12:55,160 --> 01:12:55,760 PREVENT FROM DEVELOPING THOSE 1551 01:12:55,760 --> 01:12:59,880 CONDITIONS. THERE'S A BALANCE 1552 01:12:59,880 --> 01:13:00,520 BETWEEN THESE RISK FACTORS IN 1553 01:13:00,520 --> 01:13:03,120 HIS CONTRIBUTION FOR TIME 1554 01:13:03,120 --> 01:13:04,800 BECAUSE WE KNOW THE ALS RISK 1555 01:13:04,800 --> 01:13:07,280 INCREASES AS TIME GOES ON AS 1556 01:13:07,280 --> 01:13:08,400 SOMEONE AGES. THIS 1557 01:13:08,400 --> 01:13:08,960 CONTRIBUTIONS FROM EPIC 1558 01:13:08,960 --> 01:13:11,040 GENETICS IS THE WAY THAT DNA 1559 01:13:11,040 --> 01:13:13,720 IS MAPPED. THEY CAN EITHER BE 1560 01:13:13,720 --> 01:13:14,680 INHERITED OR TRANSMITTED FROM 1561 01:13:14,680 --> 01:13:17,040 ENVIRONMENTAL CHANGES. AND 1562 01:13:17,040 --> 01:13:17,640 FINALLY, THERE'S LIFETIME 1563 01:13:17,640 --> 01:13:18,280 ENVIRONMENTS. HOW DO THEY FIT 1564 01:13:18,280 --> 01:13:24,240 INTO THIS ISSUE VERSUS 1565 01:13:24,240 --> 01:13:25,440 RESILIENCE FACTORS. 1566 01:13:25,440 --> 01:13:26,440 UNDERSTANDING WHY DO SOME 1567 01:13:26,440 --> 01:13:28,040 PEOPLE DEVELOPED ALS, SOME 1568 01:13:28,040 --> 01:13:29,320 FROM TEMPORAL DEMENTIA, AND 1569 01:13:29,320 --> 01:13:31,120 SOME BOTH AND WHY SOME NOTES 1570 01:13:31,120 --> 01:13:31,840 NEVER DEVELOP EITHER, EVEN 1571 01:13:31,840 --> 01:13:33,720 WITH THE SAME GENETIC CAUSE IS 1572 01:13:33,720 --> 01:13:35,960 CRUCIAL. WE ALSO NEED TO 1573 01:13:35,960 --> 01:13:40,280 UNDERSTAND ALS ONSET THAT WILL 1574 01:13:40,280 --> 01:13:43,080 FEED DIRECTLY INTO 1575 01:13:43,080 --> 01:13:45,640 UNDERSTANDING UNDERSTANDING 1576 01:13:45,640 --> 01:13:51,480 ALS INTO GENE CARRIERS. AND 1577 01:13:51,480 --> 01:13:52,720 THIS IS A RISK FACTOR THAT'S 1578 01:13:52,720 --> 01:13:54,680 NOT MODIFIABLE WITH 1579 01:13:54,680 --> 01:13:56,840 MANIPULATION SOME KIND OF 1580 01:13:56,840 --> 01:13:59,600 THERAPY THEN WHEN IT'S CAUSED 1581 01:13:59,600 --> 01:14:05,520 ALS BUT IT'S IMPORTANT TO HAVE 1582 01:14:05,520 --> 01:14:10,640 THE NEXT SLIDE PLEASE? DO 1583 01:14:10,640 --> 01:14:14,760 UNCOVER NEW DISEASE MECHANISMS 1584 01:14:14,760 --> 01:14:17,680 ALS THIS INCREASES OUR 1585 01:14:17,680 --> 01:14:18,520 UNDERSTANDING OF ALS. FOR 1586 01:14:18,520 --> 01:14:19,920 EXAMPLE IF WE LOOKED AT 1587 01:14:19,920 --> 01:14:21,160 FILAMENTS FOR 20 OR 30 YEARS 1588 01:14:21,160 --> 01:14:22,800 AGO WE MIGHT'VE HAD THIS IDEA 1589 01:14:22,800 --> 01:14:24,840 THAT NEURAL FILAMENTS WOULD BE 1590 01:14:24,840 --> 01:14:28,160 IMPORTANT TO MEASURE THE 1591 01:14:28,160 --> 01:14:28,720 ACTIVITY THEY HAVE TODAY. 1592 01:14:28,720 --> 01:14:35,880 THEY DID NOT EXIST. THEY CAN 1593 01:14:35,880 --> 01:14:39,400 MEASURE AN UNDERSTANDING HOW 1594 01:14:39,400 --> 01:14:46,400 NEW TECHNOLOGIES CAN ALS IS 1595 01:14:46,400 --> 01:14:47,240 VITAL WE NEED TO MAKE IT 1596 01:14:47,240 --> 01:14:48,160 EASIER TO COLLABORATE FOR 1597 01:14:48,160 --> 01:14:51,200 PEOPLE THAT NEED TO KNOW HOW 1598 01:14:51,200 --> 01:14:51,760 TO USE THESE TECHNOLOGIES WE 1599 01:14:51,760 --> 01:14:52,720 CONSTRAINTS RESEARCHERS AND 1600 01:14:52,720 --> 01:14:56,440 NEW TECHNOLOGIES. THAT'S THE 1601 01:14:56,440 --> 01:14:58,040 END OF THE PRESENTATION SO I'M 1602 01:14:58,040 --> 01:15:01,600 GOING TO HAND OVER TO MY 1603 01:15:01,600 --> 01:15:01,880 COLLEAGUE. 1604 01:15:01,880 --> 01:15:03,240 >>: THANK YOU VERY MUCH I 1605 01:15:03,240 --> 01:15:07,120 WOULD LIKE TO TALK ABOUT THE 1606 01:15:07,120 --> 01:15:07,680 WORKING GROUP TO JOIN US FOR 1607 01:15:07,680 --> 01:15:09,440 THIS CONVERSATION AND TO 1608 01:15:09,440 --> 01:15:17,760 RETURN YOUR CAMERAS. 1609 01:15:17,760 --> 01:15:18,360 [INDISTINCT] I'M GOING TO CALL 1610 01:15:18,360 --> 01:15:22,000 YOUR NAMES. 1611 01:15:22,000 --> 01:15:25,400 >>: HELLO I AM SAMMY AND 1612 01:15:25,400 --> 01:15:25,880 I'M A NEUROLOGIST AND 1613 01:15:25,880 --> 01:15:27,200 NEUROSCIENTIST AT THE 1614 01:15:27,200 --> 01:15:31,440 UNIVERSITY OF MICHIGAN. I SEE 1615 01:15:31,440 --> 01:15:33,200 PEOPLE IN THE COGNITIVE 1616 01:15:33,200 --> 01:15:33,800 DISORDER CLINIC THAT FOCUS ON 1617 01:15:33,800 --> 01:15:34,720 THIS AND JUST AS THIS WAS 1618 01:15:34,720 --> 01:15:36,680 BROUGHT UP BEFORE THERE'S A 1619 01:15:36,680 --> 01:15:37,440 GREAT DEAL OF OVERLAP BETWEEN 1620 01:15:37,440 --> 01:15:38,040 FRONTOTEMPORAL DEMENTIA IT IS 1621 01:15:38,040 --> 01:15:41,480 AND ALS AND THAT'S WHAT 1622 01:15:41,480 --> 01:15:42,520 RESEARCH FOCUSES ON. IT'S A 1623 01:15:42,520 --> 01:15:46,760 REAL PLEASURE TO BE HERE 1624 01:15:46,760 --> 01:15:48,560 TODAY. 1625 01:15:48,560 --> 01:15:49,280 >>: THANK YOU. 1626 01:15:49,280 --> 01:15:49,880 >>: GOOD MORNING EVERYONE 1627 01:15:49,880 --> 01:15:53,760 AND THANK YOU FOR HAVING ME. 1628 01:15:53,760 --> 01:15:54,840 MY NAME IS SALLY AND I AM THE 1629 01:15:54,840 --> 01:15:58,400 NEW ASSISTANT PROFESSOR AT 1630 01:15:58,400 --> 01:16:03,080 MONTRÉAL AND I STUDIED THE MIX 1631 01:16:03,080 --> 01:16:05,160 OF ALS AND I'M HAPPY TO ANSWER 1632 01:16:05,160 --> 01:16:05,680 ANY QUESTIONS HERE TODAY. 1633 01:16:05,680 --> 01:16:10,080 THANK YOU VERY MUCH. 1634 01:16:10,080 --> 01:16:11,720 >>: GOOD MORNING 1635 01:16:11,720 --> 01:16:13,840 EVERYONE, I AM A 1636 01:16:13,840 --> 01:16:16,720 NEUROSCIENTIST AT JOHN HOPKINS 1637 01:16:16,720 --> 01:16:20,840 UNIVERSITY AND THE DIRECTOR OF 1638 01:16:20,840 --> 01:16:21,320 [INDISTINCT] WHICH IS A 1639 01:16:21,320 --> 01:16:21,920 CLINICAL RESEARCH INTEGRATED 1640 01:16:21,920 --> 01:16:24,280 PROGRAM I'M ALSO GRATEFUL AND 1641 01:16:24,280 --> 01:16:26,480 HONORED TO BE HERE TODAY WITH 1642 01:16:26,480 --> 01:16:27,600 MANY PEOPLE COMING FROM ALL 1643 01:16:27,600 --> 01:16:33,480 WALKS OF LIFE. I'M ALSO 1644 01:16:33,480 --> 01:16:38,480 APPROACHING WITH HOPE FOR THE 1645 01:16:38,480 --> 01:16:39,280 FUTURE. 1646 01:16:39,280 --> 01:16:42,680 >>: I WOULD LIKE TO ECHO, 1647 01:16:42,680 --> 01:16:43,280 SINCE I AM VERY GRATEFUL FOR 1648 01:16:43,280 --> 01:16:46,280 THE OPPORTUNITY FOR 1649 01:16:46,280 --> 01:16:46,880 PARTICIPATING IN THIS WORKING 1650 01:16:46,880 --> 01:16:50,760 GROUP AND HIS EFFORTS. I AM A 1651 01:16:50,760 --> 01:16:51,520 PLEDGE OF THE COLUMBIA OF 1652 01:16:51,520 --> 01:16:53,720 UNIVERSITY AND THE DEPARTMENT 1653 01:16:53,720 --> 01:16:54,680 OF NEUROLOGY AND AT THE YORK 1654 01:16:54,680 --> 01:16:55,560 GENOME CENTER WHERE I DIRECT 1655 01:16:55,560 --> 01:16:58,440 THE CENTER FOR GENOMICS AND 1656 01:16:58,440 --> 01:17:00,320 NEURAL GENERATIVE DISEASE. IN 1657 01:17:00,320 --> 01:17:01,640 THE BASIC RESEARCHER WHO WORKS 1658 01:17:01,640 --> 01:17:03,000 ON ALS AND I ALSO SUPPORT A 1659 01:17:03,000 --> 01:17:07,200 LARGE INTERNATIONAL 1660 01:17:07,200 --> 01:17:11,440 CONSORTIUM. 1661 01:17:11,440 --> 01:17:15,680 >>: YES, MOVE YOUR 1662 01:17:15,680 --> 01:17:21,760 CAMERA. 1663 01:17:21,760 --> 01:17:22,360 >>: I GUESS I'M CURSED OR 1664 01:17:22,360 --> 01:17:27,800 SOMETHING. I MET YOU AND I AT 1665 01:17:27,800 --> 01:17:29,960 THE PROGRAM HERE MY WORK IS 1666 01:17:29,960 --> 01:17:30,640 ALL COMPUTATIONAL WORK WITH 1667 01:17:30,640 --> 01:17:31,200 INTRACRANIAL RECORDINGS AND 1668 01:17:31,200 --> 01:17:36,800 NOW I HAVE [INDISTINCT]. IN 1669 01:17:36,800 --> 01:17:39,800 LOOKING AT DISEASE AND 1670 01:17:39,800 --> 01:17:40,400 [INDISTINCT] AS EVERYONE ELSE 1671 01:17:40,400 --> 01:17:41,680 THAT IS VERY GRATEFUL TO BE 1672 01:17:41,680 --> 01:17:42,600 HERE IN A VERY GRATEFUL FOR 1673 01:17:42,600 --> 01:17:49,480 THE BRILLIANCE OF THIS PANEL. 1674 01:17:49,480 --> 01:17:52,120 >>: HELLO I AM AT THE 1675 01:17:52,120 --> 01:17:52,720 MAYO CLINIC IN JACKSONVILLE, 1676 01:17:52,720 --> 01:17:55,320 FLORIDA. I AM A PROFESSOR. 1677 01:17:55,320 --> 01:17:57,240 MY PROGRAM FOCUSES ON 1678 01:17:57,240 --> 01:17:57,920 UNDERSTANDING THE MECHANISMS 1679 01:17:57,920 --> 01:18:02,880 ASSOCIATED WITH ALS AS WELL AS 1680 01:18:02,880 --> 01:18:03,440 HEAVY EMPHASIS ON DEVELOPING 1681 01:18:03,440 --> 01:18:09,400 NOVEL BIOMARKERS FOR THE 1682 01:18:09,400 --> 01:18:11,440 DISEASE. I ECHO MY COLLECTS 1683 01:18:11,440 --> 01:18:12,200 AND IT'S A PRIVILEGE TO BE 1684 01:18:12,200 --> 01:18:12,800 WORKING ON THE RECOMMENDATIONS 1685 01:18:12,800 --> 01:18:16,040 AND PRIORITIES AND PRESENTING 1686 01:18:16,040 --> 01:18:18,040 ANSWERING ANY QUESTIONS IN 1687 01:18:18,040 --> 01:18:24,120 TODAY'S SESSION. 1688 01:18:24,120 --> 01:18:24,680 >>: I MICHAEL WARD I'M A 1689 01:18:24,680 --> 01:18:26,480 NEUROLOGIST INVESTIGATOR AT 1690 01:18:26,480 --> 01:18:27,080 THE NIH IN BETHESDA, MARYLAND. 1691 01:18:27,080 --> 01:18:31,080 I SEE PATIENTS WITH SPECTRUM 1692 01:18:31,080 --> 01:18:33,120 DISORDERS IN CLINIC AND LEAD A 1693 01:18:33,120 --> 01:18:34,200 RESEARCH TEAM INTO 1694 01:18:34,200 --> 01:18:36,320 UNDERSTANDING WHAT THE BASIC 1695 01:18:36,320 --> 01:18:37,560 MECHANISMS ARE BEHIND DISEASE. 1696 01:18:37,560 --> 01:18:38,000 I ALSO CO- LEAD THE 1697 01:18:38,000 --> 01:18:39,800 NEURODEGENERATIVE DISEASE 1698 01:18:39,800 --> 01:18:43,400 INITIATIVE HERE WHICH IS A 1699 01:18:43,400 --> 01:18:45,000 PUBLIC, PRIVATE PARTNERSHIP TO 1700 01:18:45,000 --> 01:18:45,600 MAKE A LARGE REPOSITORY OF 1701 01:18:45,600 --> 01:18:49,440 STEM CELL MODELS OF ALS AND TO 1702 01:18:49,440 --> 01:18:50,160 SHARE WITH THE BROAD RESEARCH 1703 01:18:50,160 --> 01:18:50,760 COMMUNITY. I'M DELIGHTED TO 1704 01:18:50,760 --> 01:18:57,200 BE HERE TODAY. I WANT TO 1705 01:18:57,200 --> 01:18:58,720 THANK ALL OF YOU WOULD FOR 1706 01:18:58,720 --> 01:18:59,320 YOUR WORK IN THE INTERSECTION 1707 01:18:59,320 --> 01:19:01,160 OF THIS PLAN. WE HAVE HAD ANY 1708 01:19:01,160 --> 01:19:03,400 COMMUNITY DISCUSSIONS THAT 1709 01:19:03,400 --> 01:19:07,520 HAVE LED TO PRIORITIES OF THAT 1710 01:19:07,520 --> 01:19:09,440 WERE JUST DESCRIBED. IN THE 1711 01:19:09,440 --> 01:19:10,720 FROM THE NEXT 15 MINUTES WERE 1712 01:19:10,720 --> 01:19:16,240 TO DISCUSS THE DISEASES AND 1713 01:19:16,240 --> 01:19:23,920 [INDISTINCT] ONE OF THE FIRST 1714 01:19:23,920 --> 01:19:27,520 THINGS THAT PROMOTING 1715 01:19:27,520 --> 01:19:28,080 COLLABORATIONS BETWEEN BASIC 1716 01:19:28,080 --> 01:19:32,080 AND CLEAN RESEARCHERS IS 1717 01:19:32,080 --> 01:19:33,640 REALLY A CRUCIAL STEP TO OUR 1718 01:19:33,640 --> 01:19:34,680 ADVANCE UNDERSTANDING 1719 01:19:34,680 --> 01:19:36,320 MECHANISMS WITH ALS. AND SO 1720 01:19:36,320 --> 01:19:36,880 MANY OF THE PANEL LIKE TO 1721 01:19:36,880 --> 01:19:45,280 ELABORATE ON THIS? 1722 01:19:45,280 --> 01:19:45,800 >>: WE ARE IN NEED OF 1723 01:19:45,800 --> 01:19:50,960 THIS COOPERATION AND TO 1724 01:19:50,960 --> 01:19:53,720 SUMMARIZE WILLIE THIS IS A 1725 01:19:53,720 --> 01:19:55,840 DISEASE FOR WHAT WE WILL BE 1726 01:19:55,840 --> 01:19:58,760 DOING BECAUSE WE KNOW THE 1727 01:19:58,760 --> 01:19:59,640 DISEASE IS HETEROGENEOUS, BOTH 1728 01:19:59,640 --> 01:20:02,640 SPORADIC NOT FAMILIAL AND 1729 01:20:02,640 --> 01:20:10,800 UNFAMILIAR FORMS. AND IN THIS 1730 01:20:10,800 --> 01:20:15,720 IS INDIFFERENT TO PATIENTS AND 1731 01:20:15,720 --> 01:20:16,520 THIS IS DIFFERENT ET CETERA. 1732 01:20:16,520 --> 01:20:17,160 SO FOR US TO UNDERSTAND WHAT 1733 01:20:17,160 --> 01:20:19,120 ARE THE CAUSES UNDERLINED HIS 1734 01:20:19,120 --> 01:20:24,320 THESE DIFFERENCES AND WE NEED 1735 01:20:24,320 --> 01:20:25,680 ORGANIC AND FREQUENT 1736 01:20:25,680 --> 01:20:26,640 COMMUNICATION BETWEEN THE 1737 01:20:26,640 --> 01:20:27,720 RESEARCHERS INTO THE 1738 01:20:27,720 --> 01:20:28,320 CLINICIANS CAN TELL US HOW THE 1739 01:20:28,320 --> 01:20:28,840 PATIENTS HAVE A SPECIFIC 1740 01:20:28,840 --> 01:20:31,480 MUTATION THAT CAN MANIFEST IN 1741 01:20:31,480 --> 01:20:32,600 PROGRESS. SO WHEN LOOKING FOR 1742 01:20:32,600 --> 01:20:38,480 THE GENES AND THE SPECIFIC 1743 01:20:38,480 --> 01:20:40,440 GROUP OF PATIENTS AND WE KNOW 1744 01:20:40,440 --> 01:20:41,600 EXACTLY WHAT MAKES THE 1745 01:20:41,600 --> 01:20:42,200 PATIENTS WHO THEY ARE. AND WE 1746 01:20:42,200 --> 01:20:46,040 NEED AN ORGANIC COMMUNICATION 1747 01:20:46,040 --> 01:20:48,320 AND THE BASIS OF THE FINDINGS 1748 01:20:48,320 --> 01:20:55,920 FROM THE BASIC RESEARCH AND 1749 01:20:55,920 --> 01:20:56,640 THIS IS TAILORED FOR THESE 1750 01:20:56,640 --> 01:20:58,520 GROUP OF PATIENTS. I WOULD 1751 01:20:58,520 --> 01:20:59,800 LIKE TO SAY THAT THERE ARE 1752 01:20:59,800 --> 01:21:03,240 CURRENTLY ONGOING AND IN RED 1753 01:21:03,240 --> 01:21:07,080 STATES AND ELSEWHERE WERE THEY 1754 01:21:07,080 --> 01:21:08,280 TALK ABOUT THE GRADING OF THE 1755 01:21:08,280 --> 01:21:10,480 CONSORTIUM AND THE NIH NETWORK 1756 01:21:10,480 --> 01:21:15,960 IS ALS AND THE TARGET ALS BIO 1757 01:21:15,960 --> 01:21:17,120 MARKETS PROGRAM. BUT NOW 1758 01:21:17,120 --> 01:21:18,880 OUTLINING OUR PRIORITIES AND 1759 01:21:18,880 --> 01:21:19,400 OUR COMMITTEE FELT THAT WE 1760 01:21:19,400 --> 01:21:21,200 NEEDED TO STRESS THAT THERE IS 1761 01:21:21,200 --> 01:21:24,720 THE NEED TO SET THIS APPROACH 1762 01:21:24,720 --> 01:21:25,320 EVERYWHERE. THE NATURE CENTER 1763 01:21:25,320 --> 01:21:26,840 AND THEN ENCOURAGE CLINICIANS 1764 01:21:26,840 --> 01:21:28,800 TO TALK TO THE RESEARCHERS AND 1765 01:21:28,800 --> 01:21:34,600 VICE VERSA BECAUSE THIS 1766 01:21:34,600 --> 01:21:35,160 APPROACH REALLY FORMS THE 1767 01:21:35,160 --> 01:21:36,000 BASIS FOR EFFICIENT EXECUTING 1768 01:21:36,000 --> 01:21:37,520 OF A PLAN THAT MY COLLEAGUES 1769 01:21:37,520 --> 01:21:38,200 THAT HAVE BEEN DISCUSSING IN A 1770 01:21:38,200 --> 01:21:43,120 MINUTE. IN THE DEVELOPING AND 1771 01:21:43,120 --> 01:21:44,000 ASSISTING THE TRUE MEANING OF 1772 01:21:44,000 --> 01:21:47,280 SPORADIC ALS FOR INSTANCE, AND 1773 01:21:47,280 --> 01:21:49,160 CREATE INFORMATION FOR THIS 1774 01:21:49,160 --> 01:21:50,880 DISEASE ET CETERA. WE NEED TO 1775 01:21:50,880 --> 01:21:53,000 UNDERSTAND THIS BETWEEN CLINIC 1776 01:21:53,000 --> 01:21:54,040 AND BASIC RESEARCHER IT IS 1777 01:21:54,040 --> 01:21:55,400 ROLLOUTS TO ONE: UNDERSTAND 1778 01:21:55,400 --> 01:21:59,040 THE DISEASE AND TWO: TO 1779 01:21:59,040 --> 01:22:00,080 DEVELOP TRIALS CLINICALLY THAT 1780 01:22:00,080 --> 01:22:01,520 ARE A SPECIFIC SUBGROUP OF 1781 01:22:01,520 --> 01:22:03,120 PATIENTS. I WOULD ALSO LIKE 1782 01:22:03,120 --> 01:22:08,480 TO ADD THAT WE CAN HAVE THE 1783 01:22:08,480 --> 01:22:09,240 BEST RESEARCH MODELS IN THE 1784 01:22:09,240 --> 01:22:11,440 LAB BUT IF WE DO NOT INVOLVE 1785 01:22:11,440 --> 01:22:12,120 THE PATIENTS EARLY ON AND 1786 01:22:12,120 --> 01:22:13,960 THEREFORE, BRING THE CLINICAL 1787 01:22:13,960 --> 01:22:14,560 RESEARCH I DON'T THINK WE CAN 1788 01:22:14,560 --> 01:22:18,360 GO AS FAR. 1789 01:22:18,360 --> 01:22:19,920 >>: THANK YOU. AS YOU 1790 01:22:19,920 --> 01:22:22,600 JUST SAID. WERE GOING WITH 1791 01:22:22,600 --> 01:22:24,880 GREAT CLINICAL INFORMATION. 1792 01:22:24,880 --> 01:22:27,880 GOING TO MAKE A DIFFERENCE. 1793 01:22:27,880 --> 01:22:30,760 AND IN THAT SENSE IT COULD BE 1794 01:22:30,760 --> 01:22:35,880 REALLY BROADLY SHARED AND THEY 1795 01:22:35,880 --> 01:22:39,120 LOOKING AT PLATFORMS AND MAYBE 1796 01:22:39,120 --> 01:22:49,080 DESCRIBE THE CHALLENGES YOU'VE 1797 01:22:49,080 --> 01:22:51,800 HEARD TODAY THAT ALX IS A 1798 01:22:51,800 --> 01:22:52,400 COMPLEX DISEASE IS CLINICALLY 1799 01:22:52,400 --> 01:22:54,040 HETEROGENEOUS IN THE MANIFEST 1800 01:22:54,040 --> 01:22:59,320 DIFFERENTLY AND ALSO 1801 01:22:59,320 --> 01:23:00,720 GENETICALLY COMPLEX. YOU CAN 1802 01:23:00,720 --> 01:23:01,840 HAVE MUTATIONS THAT ARE RARE 1803 01:23:01,840 --> 01:23:02,880 AND HAVE A LARGE REFLECT AND 1804 01:23:02,880 --> 01:23:04,720 THEN HAVE SEVERAL MUTATIONS 1805 01:23:04,720 --> 01:23:05,720 THAT COMBINE AND EACH OF THEM 1806 01:23:05,720 --> 01:23:10,040 HAVE SMALL EFFECTS. IT IS 1807 01:23:10,040 --> 01:23:10,640 ALSO A RELATIVELY RARE DISEASE 1808 01:23:10,640 --> 01:23:13,200 AND IN ORDER TO GET THE LARGE 1809 01:23:13,200 --> 01:23:16,720 NUMBER OF SAMPLE SIZES THAT 1810 01:23:16,720 --> 01:23:19,280 YOU NEED FOR PEOPLE TO WORK 1811 01:23:19,280 --> 01:23:21,800 TOGETHER TO COMBINE THESE 1812 01:23:21,800 --> 01:23:24,000 SAMPLES NEED TO BRIDGE THE 1813 01:23:24,000 --> 01:23:28,440 PARTNERSHIP OF DIFFERENT 1814 01:23:28,440 --> 01:23:33,000 CENTERS. AND WE CAN TALK 1815 01:23:33,000 --> 01:23:34,520 ABOUT EVERYBODY STUDIES THESE 1816 01:23:34,520 --> 01:23:36,080 DIFFERENTLY. IF HE WANTED TO 1817 01:23:36,080 --> 01:23:37,760 STUDY THE RELATIONSHIP BETWEEN 1818 01:23:37,760 --> 01:23:39,240 HOW A PARTICULAR BIOMARKER 1819 01:23:39,240 --> 01:23:40,760 CORRELATES WITH A PARTICULAR 1820 01:23:40,760 --> 01:23:42,280 TYPE OF DISEASE PROGRESSION, 1821 01:23:42,280 --> 01:23:43,520 AND EMPLOY NEW TECHNOLOGIES TO 1822 01:23:43,520 --> 01:23:45,720 STUDY THESE BIO SAMPLES FROM 1823 01:23:45,720 --> 01:23:46,920 NATIONS LIKE THESE, AND THEN 1824 01:23:46,920 --> 01:23:49,040 HOW THIS RELATES TO THEIR 1825 01:23:49,040 --> 01:23:50,040 GENOTYPE WE NEED TO BE ABLE TO 1826 01:23:50,040 --> 01:23:50,640 COLLECT ALL OF THE INFORMATION 1827 01:23:50,640 --> 01:23:56,120 AND ALL OF THE SAMPLES IN A 1828 01:23:56,120 --> 01:23:57,080 WAY THAT YOU CAN COMPARE 1829 01:23:57,080 --> 01:23:58,480 ACROSS DIFFERENT SCIENCE. IN 1830 01:23:58,480 --> 01:24:00,440 THE LAST EIGHT-TEN-YEARS THERE 1831 01:24:00,440 --> 01:24:02,080 HAS BEEN A SEA CHANGE IN HOW 1832 01:24:02,080 --> 01:24:03,120 WE HAVE APPROACHED THIS IN THE 1833 01:24:03,120 --> 01:24:07,120 ALS COMMUNITY. THERE'S ABOUT 1834 01:24:07,120 --> 01:24:09,760 HALF A DEGEN LARGE, MULTI- 1835 01:24:09,760 --> 01:24:11,280 CENTER AND MULTINATIONAL 1836 01:24:11,280 --> 01:24:13,120 EFFORTS. WE'VE LEARNED A LOT 1837 01:24:13,120 --> 01:24:14,440 THROUGH DOING THIS ABOUT WHAT 1838 01:24:14,440 --> 01:24:16,080 IT TAKES FOR WHAT IS THE 1839 01:24:16,080 --> 01:24:16,720 MINIMUM AMOUNT OF INFORMATION 1840 01:24:16,720 --> 01:24:19,120 THAT YOU NEED, AND HOW IT 1841 01:24:19,120 --> 01:24:20,520 NEEDS TO BE COLLECTED AND HOW 1842 01:24:20,520 --> 01:24:31,000 IT NEEDS TO BE PUT TOGETHER. 1843 01:24:35,640 --> 01:24:36,640 >>: WE LOST TO EMILY IN 1844 01:24:36,640 --> 01:24:37,080 YOUR LAST SENTENCE. 1845 01:24:37,080 --> 01:24:39,280 >>: IS BETTER? 1846 01:24:39,280 --> 01:24:39,720 >>: MUCH BETTER. 1847 01:24:39,720 --> 01:24:43,040 >>: THANK YOU. 1848 01:24:43,040 --> 01:24:43,720 >>: WE NEED TO 1849 01:24:43,720 --> 01:24:44,160 INCORPORATE ALL THIS 1850 01:24:44,160 --> 01:24:44,720 INFORMATION TO THE QUESTIONS 1851 01:24:44,720 --> 01:24:50,480 THAT WE ARE ASKING. IT'S ONE 1852 01:24:50,480 --> 01:24:51,520 THING TO KNOW THAT CITIES LIKE 1853 01:24:51,520 --> 01:24:54,960 THEM ARE HAPPENING AND IT'S 1854 01:24:54,960 --> 01:24:58,720 ANOTHER TO MAKE THIS 1855 01:24:58,720 --> 01:24:59,120 INFORMATION WIDELY 1856 01:24:59,120 --> 01:25:00,280 DISSEMINATED. THE LARGE 1857 01:25:00,280 --> 01:25:01,840 STUDIES THAT WE KNOW UP TO 1858 01:25:01,840 --> 01:25:03,280 EACH OTHER AND WORKED OUT WAYS 1859 01:25:03,280 --> 01:25:04,720 TO SHARE INFORMATION THE NEXT 1860 01:25:04,720 --> 01:25:08,960 CHALLENGE BEFORE US AND HOW TO 1861 01:25:08,960 --> 01:25:09,720 INCORPORATE OTHER UNDERSTUDIED 1862 01:25:09,720 --> 01:25:11,640 GROUPS AND TO BUILD ON WHAT 1863 01:25:11,640 --> 01:25:12,280 WE'VE LEARNED SO FAR ABOUT 1864 01:25:12,280 --> 01:25:16,160 DOING A LARGE MULTINATIONAL 1865 01:25:16,160 --> 01:25:17,720 EFFORTS. 1866 01:25:17,720 --> 01:25:19,440 >>: THANK YOU SO MUCH. 1867 01:25:19,440 --> 01:25:20,040 IT'S A GREAT SEGUE TO THE NEXT 1868 01:25:20,040 --> 01:25:24,920 QUESTION I WANTED TO PUT UP 1869 01:25:24,920 --> 01:25:26,640 HERE. THIS IS THE ASPECT THAT 1870 01:25:26,640 --> 01:25:28,520 WE WANTED TO HIGHLIGHT 1871 01:25:28,520 --> 01:25:32,000 PRIORITIES AND THIS FREQUENTLY 1872 01:25:32,000 --> 01:25:33,520 COMES UP WITH CORRECT 1873 01:25:33,520 --> 01:25:34,840 KNOWLEDGE AND IS BASED ON DATA 1874 01:25:34,840 --> 01:25:35,640 FROM PEOPLE OF EUROPEAN 1875 01:25:35,640 --> 01:25:39,600 ANCESTRY AND THEN FOR PEOPLE 1876 01:25:39,600 --> 01:25:43,480 WITH YEAST EUROPEAN ANCESTRY. 1877 01:25:43,480 --> 01:25:45,440 IT'S NOT SPECIFIC TO ALS 1878 01:25:45,440 --> 01:25:45,960 RESEARCH FOR OUR COMMUNITY 1879 01:25:45,960 --> 01:25:49,560 NEEDS THIS TO CHANGE AND 1880 01:25:49,560 --> 01:25:50,960 RESEARCH ACROSS ANCESTRIES. 1881 01:25:50,960 --> 01:25:51,440 SO SALLY WOULD YOU MIND 1882 01:25:51,440 --> 01:25:54,080 DESCRIBING THIS ISSUE AND THEN 1883 01:25:54,080 --> 01:25:57,840 TALK ABOUT ALS RESEARCH. 1884 01:25:57,840 --> 01:25:58,800 >>: ABSOLUTELY BEFORE I 1885 01:25:58,800 --> 01:26:00,400 ANSWER THIS QUESTION I WILL 1886 01:26:00,400 --> 01:26:02,440 ADDRESS WHY THIS EVENT HAPPENS 1887 01:26:02,440 --> 01:26:03,320 BECAUSE CERTAINLY WHEN YOU GET 1888 01:26:03,320 --> 01:26:04,960 THE DATA SET WE DO CLINICIANS 1889 01:26:04,960 --> 01:26:07,080 ARE NOT ONLY SEEING THOSE OF 1890 01:26:07,080 --> 01:26:13,400 EUROPEAN ANCESTRY. WHAT 1891 01:26:13,400 --> 01:26:15,200 HAPPENS IS CLINICIANS COLLECT 1892 01:26:15,200 --> 01:26:18,160 ALL THE DATA. THEY SAY ARE 1893 01:26:18,160 --> 01:26:20,320 YOU EUROPEAN * EUROPEAN 1894 01:26:20,320 --> 01:26:22,520 ORANGE? WHAT HAPPENS AT THE 1895 01:26:22,520 --> 01:26:24,680 GENETIC LEVEL WHEN YOU GO TO 1896 01:26:24,680 --> 01:26:25,280 IMPUTE GENETIC INFORMATION AND 1897 01:26:25,280 --> 01:26:27,120 FILE ALL OF THESE POPULATIONS 1898 01:26:27,120 --> 01:26:32,520 ACROSS THE MAP YOU SEE THE 1899 01:26:32,520 --> 01:26:34,160 MAJORITY OF PARTICIPANTS 1900 01:26:34,160 --> 01:26:35,360 THEMSELVES THAT ARE JUST BEING 1901 01:26:35,360 --> 01:26:36,000 IN BASIC RESEARCH STUDIES TEND 1902 01:26:36,000 --> 01:26:39,640 TO BE OF EUROPEAN DESCENT. 1903 01:26:39,640 --> 01:26:40,640 THERE'S A LOT OF DIFFERENT 1904 01:26:40,640 --> 01:26:43,080 STUDIES AS TO WHY THAT HAPPENS 1905 01:26:43,080 --> 01:26:45,840 AS WELL. BUT WHAT HAPPENS AT 1906 01:26:45,840 --> 01:26:47,840 THE GENETIC LEVEL IS WHEN 1907 01:26:47,840 --> 01:26:48,720 YOU'RE TRYING TO MAKE A 1908 01:26:48,720 --> 01:26:49,360 HOMOGENEOUS POPULATION BECAUSE 1909 01:26:49,360 --> 01:26:52,120 YOU WANT TO COMPARE APPLES TO 1910 01:26:52,120 --> 01:26:54,360 APPLES. SO YOU CAN KEEP IT 1911 01:26:54,360 --> 01:26:58,440 SIMPLE THIS WAY. IF YOU ARE 1912 01:26:58,440 --> 01:26:59,120 INCORPORATING DIFFERENT 1913 01:26:59,120 --> 01:27:00,360 ETHNICITIES FOR EXAMPLE, THOSE 1914 01:27:00,360 --> 01:27:02,760 FROM SOUTH AMERICA OR AFRICA, 1915 01:27:02,760 --> 01:27:04,720 YOU'RE GOING TO SEE A SIGNAL 1916 01:27:04,720 --> 01:27:07,840 THAT IS ENRICHED AND CASES AND 1917 01:27:07,840 --> 01:27:08,400 THIS IS GOING TO BE 1918 01:27:08,400 --> 01:27:09,000 STATISTICALLY SIGNIFICANT AND 1919 01:27:09,000 --> 01:27:12,200 WILL ALLOW THE RESEARCHER TO 1920 01:27:12,200 --> 01:27:16,320 NAÏVELY CONCLUDE THAT THAT 1921 01:27:16,320 --> 01:27:17,720 SPECIFIC STATISTICALLY 1922 01:27:17,720 --> 01:27:18,280 SIGNIFICANT SIGNAL COULD BE 1923 01:27:18,280 --> 01:27:20,880 EXPLOITING THE DISEASE. BUT 1924 01:27:20,880 --> 01:27:23,000 REALLY THAT SIGNAL IS 1925 01:27:23,000 --> 01:27:27,400 ORIGINATING FROM GEOGRAPHICAL 1926 01:27:27,400 --> 01:27:28,600 BARRIERS AND DIFFERENT 1927 01:27:28,600 --> 01:27:29,960 ANCESTRAL INFORMATION. THIS 1928 01:27:29,960 --> 01:27:31,160 HAS A HUGE CONSEQUENCE IF YOU 1929 01:27:31,160 --> 01:27:35,400 DO NOT KEEP YOUR POPULATION 1930 01:27:35,400 --> 01:27:39,920 HOMOGENEOUS. [INDISTINCT] 1931 01:27:39,920 --> 01:27:44,800 >>: WE JUST LOST YOU. 1932 01:27:44,800 --> 01:27:45,400 >>: OH SORRY, CAN'T HEAR 1933 01:27:45,400 --> 01:27:45,680 HEAR ME NOW. 1934 01:27:45,680 --> 01:27:48,720 >>: YES WE CAN. 1935 01:27:48,720 --> 01:27:54,560 >>: SO THERE ARE 1936 01:27:54,560 --> 01:27:56,320 UNFORTUNATELY, NOT COMMENTS 1937 01:27:56,320 --> 01:27:57,720 ALS IS HAPPENS ACROSS ALL 1938 01:27:57,720 --> 01:28:00,320 DISEASES. WE ARE ABLE TO 1939 01:28:00,320 --> 01:28:01,760 LEARN DIFFERENT GROUPS THAT 1940 01:28:01,760 --> 01:28:04,280 HAVE BEEN REALLY WORKING TO 1941 01:28:04,280 --> 01:28:07,160 FIX THIS ISSUE. THIS 1942 01:28:07,160 --> 01:28:07,560 PSYCHIATRIC GENOMICS 1943 01:28:07,560 --> 01:28:09,560 ASSOCIATION FOR EXAMPLE HAS 1944 01:28:09,560 --> 01:28:10,920 REALLY GOOD ESTABLISHMENTS 1945 01:28:10,920 --> 01:28:12,480 FROM EAST ASIA, SOUTH AMERICA, 1946 01:28:12,480 --> 01:28:13,560 SOUTH AFRICA AND EAST AFRICA 1947 01:28:13,560 --> 01:28:14,560 THEY'VE BEEN ABLE TO PUT BEST 1948 01:28:14,560 --> 01:28:19,640 PRACTICES IN PLACE HERE. 1949 01:28:19,640 --> 01:28:21,440 TYPICALLY WHAT YOU CAN DO IS 1950 01:28:21,440 --> 01:28:22,000 CREATE SMALLER SUBSETS OF 1951 01:28:22,000 --> 01:28:25,520 MATCHING ANCESTRIES SO THAT 1952 01:28:25,520 --> 01:28:27,080 YOU CAN EITHER DO A COMBINED 1953 01:28:27,080 --> 01:28:29,120 META-ANALYSIS OF THE EVERYBODY 1954 01:28:29,120 --> 01:28:31,520 IS INCLUDED AND YOU ARE 1955 01:28:31,520 --> 01:28:33,440 ACCOUNTING FOR THESE 1956 01:28:33,440 --> 01:28:33,840 STATISTICAL FALSE 1957 01:28:33,840 --> 01:28:34,720 ASSOCIATIONS, OR YOU COULD 1958 01:28:34,720 --> 01:28:36,040 CREATE A BETTER SOFTWARE 1959 01:28:36,040 --> 01:28:37,960 PROGRAMS THAT ARE ABLE TO 1960 01:28:37,960 --> 01:28:42,560 ACCOUNT FOR THESE VERY COMMON 1961 01:28:42,560 --> 01:28:44,320 STATISTICAL ARTIFACTS AS WELL. 1962 01:28:44,320 --> 01:28:44,960 BOTH EFFORTS ARE HAPPENING, 1963 01:28:44,960 --> 01:28:45,800 IT'S IMPORTANT NOT BECAUSE YOU 1964 01:28:45,800 --> 01:28:47,000 WANT TO FEEL GOOD ABOUT 1965 01:28:47,000 --> 01:28:49,320 OURSELVES THAT WE WANT TO BE 1966 01:28:49,320 --> 01:28:49,960 INCLUSIVE, BUT THIS HAS ACTUAL 1967 01:28:49,960 --> 01:28:53,720 REAL IMPLICATIONS. THE EFFECT 1968 01:28:53,720 --> 01:28:55,920 SIZE OF A SPECIFIC REEL THAT 1969 01:28:55,920 --> 01:28:57,480 MIGHT BE ASSOCIATED WITH 1970 01:28:57,480 --> 01:28:58,120 DISEASE COULD BE A LOT 1971 01:28:58,120 --> 01:29:00,040 DIFFERENT IN A CERTAIN 1972 01:29:00,040 --> 01:29:02,280 ETHNICITY THAN IT IS IN YOUR 1973 01:29:02,280 --> 01:29:05,560 PLANS FOR EXAMPLE. WE HAVE 1974 01:29:05,560 --> 01:29:06,320 EVIDENCE OF THAT WIND 1975 01:29:06,320 --> 01:29:07,040 CONDITIONS ADMINISTER UP. OH 1976 01:29:07,040 --> 01:29:07,640 AND THE UPPERCUT RIGHT THERE 1977 01:29:07,640 --> 01:29:08,520 WAS MEDICATION. IF YOU GIVE 1978 01:29:08,520 --> 01:29:10,400 THE WRONG DOSE THAT COULD HAVE 1979 01:29:10,400 --> 01:29:12,520 TREMENDOUS IMPLICATIONS FOR 1980 01:29:12,520 --> 01:29:13,920 THE PATIENTS THEMSELVES. THIS 1981 01:29:13,920 --> 01:29:15,280 IS AN ONGOING EFFORT AND I 1982 01:29:15,280 --> 01:29:18,320 WENT TO RECOGNIZE A GENETICIST 1983 01:29:18,320 --> 01:29:18,960 HERE WHO IS WORKING ON THIS. 1984 01:29:18,960 --> 01:29:24,680 HIS NAME IS [INDISTINCT] IS A 1985 01:29:24,680 --> 01:29:25,240 COLLEAGUE OF MINE AND WORKS 1986 01:29:25,240 --> 01:29:30,480 CLOSELY OF COURSE AND HE IS AT 1987 01:29:30,480 --> 01:29:33,680 KINGS COLLEGE AND HATS OFF TO 1988 01:29:33,680 --> 01:29:34,280 HIM FOR REALLY BRINGING IN 1989 01:29:34,280 --> 01:29:34,720 UNDERREPRESENTED THESE 1990 01:29:34,720 --> 01:29:38,560 COMMUNITIES AND HE HIMSELF IS 1991 01:29:38,560 --> 01:29:41,120 THE RESEARCHER AND HAS BROUGHT 1992 01:29:41,120 --> 01:29:44,280 IN GROUPS FROM TURKEY, 1993 01:29:44,280 --> 01:29:45,240 ETHIOPIA, INDIA, FROM ISRAEL, 1994 01:29:45,240 --> 01:29:46,560 OTHER PARTS OF THE MIDDLE EAST 1995 01:29:46,560 --> 01:29:48,800 AS WELL AND HIS GOAL IS TO 1996 01:29:48,800 --> 01:29:51,880 MAKE HIMSELF, AND ALL OF US 1997 01:29:51,880 --> 01:29:52,400 AVAILABLE IN ADVANCING ALS 1998 01:29:52,400 --> 01:29:55,200 GENETICS RESEARCH ACROSS 1999 01:29:55,200 --> 01:29:55,760 DIFFERENT COUNTRIES THAT ARE 2000 01:29:55,760 --> 01:30:00,040 NOT REPRESENTED AS WELL. THIS 2001 01:30:00,040 --> 01:30:01,080 IS AN ONGOING EFFORT AND THIS 2002 01:30:01,080 --> 01:30:03,080 HAS VERY CLEAR IMPLICATIONS 2003 01:30:03,080 --> 01:30:07,120 AND STAY TUNED AS OUR DATA 2004 01:30:07,120 --> 01:30:10,200 BECOMES MORE DIVERSE AND WE 2005 01:30:10,200 --> 01:30:12,960 LEARN ABOUT THE EFFECTS THAT 2006 01:30:12,960 --> 01:30:13,840 WE CAN SEE IN THE EUROPEAN 2007 01:30:13,840 --> 01:30:15,280 POPULATION AS WELL AS NEW ONES 2008 01:30:15,280 --> 01:30:20,200 THAT WE HAVE UNVEILED AS WELL. 2009 01:30:20,200 --> 01:30:21,000 >>: THANK YOU VERY MUCH. 2010 01:30:21,000 --> 01:30:26,320 OUR FIRST PRIORITY IS TO 2011 01:30:26,320 --> 01:30:27,160 ATTACK SPORADIC ALS AND WE CAN 2012 01:30:27,160 --> 01:30:28,400 ALL RECOGNIZE THIS IS AN AREA 2013 01:30:28,400 --> 01:30:31,320 WHERE WE ALL KNOW VERY LITTLE 2014 01:30:31,320 --> 01:30:31,840 AND ONE REASON FOR THAT IS 2015 01:30:31,840 --> 01:30:33,800 BECAUSE OF THE CHALLENGES IN 2016 01:30:33,800 --> 01:30:39,560 MODERATING IT AND MAYBE GLENN 2017 01:30:39,560 --> 01:30:41,640 AND SAMMY IF YOU COULD COMMENT 2018 01:30:41,640 --> 01:30:41,880 ON THAT. 2019 01:30:41,880 --> 01:30:45,040 >>: SURE. I'M HAPPY TO 2020 01:30:45,040 --> 01:30:46,520 TALK A LITTLE BIT ABOUT SOME 2021 01:30:46,520 --> 01:30:48,520 OF THE NEWER APPROACHES FOR 2022 01:30:48,520 --> 01:30:51,440 MODERATING MONITORING SPORADIC 2023 01:30:51,440 --> 01:30:53,360 ALS. I THINK IN DOING SO IT'S 2024 01:30:53,360 --> 01:30:56,760 IMPORTANT TO THINK ABOUT WHAT 2025 01:30:56,760 --> 01:30:58,160 SPORADIC DISEASE IS AND TO 2026 01:30:58,160 --> 01:30:59,600 RECOGNIZE OUR LIMITATIONS. IF 2027 01:30:59,600 --> 01:31:00,280 WE WANT TO REPRODUCE SOMETHING 2028 01:31:00,280 --> 01:31:02,160 WE NEED TO KNOW WHAT TO 2029 01:31:02,160 --> 01:31:02,760 REPRODUCE AND I THINK THAT'S 2030 01:31:02,760 --> 01:31:04,240 ONE OF THE BIGGEST CHALLENGES 2031 01:31:04,240 --> 01:31:07,200 AND SPORADIC SEES. THERE'S 2032 01:31:07,200 --> 01:31:08,240 TWO WAYS OF GOING ABOUT THIS. 2033 01:31:08,240 --> 01:31:10,520 WE CAN SAY I DON'T KNOW WHAT 2034 01:31:10,520 --> 01:31:13,880 THIS IS BUT I CAN STILL GET 2035 01:31:13,880 --> 01:31:14,720 CELLS FROM INDIVIDUALS WITH 2036 01:31:14,720 --> 01:31:15,640 SPORADIC ALS AND THOSE CAN BE 2037 01:31:15,640 --> 01:31:17,320 REPROGRAMMED INTO STEM CELLS 2038 01:31:17,320 --> 01:31:21,440 AND THOSE CAN BE MADE INTO 2039 01:31:21,440 --> 01:31:23,360 NEURONS IN THE CELL TYPE IS 2040 01:31:23,360 --> 01:31:25,480 AFFECTED AND THEN WE CAN STUDY 2041 01:31:25,480 --> 01:31:27,800 THAT IN ADDITION WE CAN TEST 2042 01:31:27,800 --> 01:31:30,400 OUT THERAPIES AND TRY TO 2043 01:31:30,400 --> 01:31:31,600 DETERMINE DISEASE MECHANISMS. 2044 01:31:31,600 --> 01:31:32,200 AND THAT IS PROVING FRUITFUL 2045 01:31:32,200 --> 01:31:35,440 IN A NUMBER OF RESPECTS BUT, 2046 01:31:35,440 --> 01:31:36,000 THE ALTERNATIVE WAY IS TO 2047 01:31:36,000 --> 01:31:40,640 ACTUALLY TRY TO IDENTIFY THE 2048 01:31:40,640 --> 01:31:41,280 UNDERLYING REASONS FOR 2049 01:31:41,280 --> 01:31:42,880 SPORADIC ALS AND I THINK A LOT 2050 01:31:42,880 --> 01:31:43,440 OF THE GENETIC STUDIES ARE 2051 01:31:43,440 --> 01:31:47,320 MAKING QUITE A BIT OF PROGRESS 2052 01:31:47,320 --> 01:31:49,720 THERE. THERE'S ALSO A LOT OF 2053 01:31:49,720 --> 01:31:51,040 INTEREST IN THE ENVIRONMENTAL 2054 01:31:51,040 --> 01:31:52,040 EXPOSURES THAT MAY CHANGE ONCE 2055 01:31:52,040 --> 01:31:56,240 RISK OF DEVELOPING ALS OVER 2056 01:31:56,240 --> 01:31:58,320 TIME. THIS IS NOT LIMITED TO 2057 01:31:58,320 --> 01:32:00,400 ALS. THIS IS SOMETHING THAT 2058 01:32:00,400 --> 01:32:02,360 WE RUN INTO A LOT WITH MANY 2059 01:32:02,360 --> 01:32:04,000 AGE-RELATED DISORDERS OF THE 2060 01:32:04,000 --> 01:32:04,680 PROBABLY EXPOSURES OVER THE 2061 01:32:04,680 --> 01:32:06,320 COURSE OF OUR LIFETIME THAT 2062 01:32:06,320 --> 01:32:07,480 AFFECT RISK, MAYBE NOT 2063 01:32:07,480 --> 01:32:08,360 IMMEDIATELY, BUT TEN-YEARS 2064 01:32:08,360 --> 01:32:10,640 DOWN THE LINE. AND IT BE VERY 2065 01:32:10,640 --> 01:32:14,160 CHALLENGING TO PARCEL THOSE 2066 01:32:14,160 --> 01:32:16,720 OUT. THIS IS AN AREA OF FOCUS 2067 01:32:16,720 --> 01:32:19,520 FOR THE NIH. IT IS ACTUALLY 2068 01:32:19,520 --> 01:32:20,080 ONE OF THE TOPICS THAT WAS 2069 01:32:20,080 --> 01:32:22,160 AWARDED A TRANSFORMATIVE 2070 01:32:22,160 --> 01:32:26,520 RESEARCH APPLICATION OR AWARD 2071 01:32:26,520 --> 01:32:27,840 FOR A GROUP HERE TO LOOK AT 2072 01:32:27,840 --> 01:32:28,400 ENVIRONMENTAL EXPOSURES AND 2073 01:32:28,400 --> 01:32:34,000 ACTUALLY THE CONNECTION WITH 2074 01:32:34,000 --> 01:32:34,680 GENETICALLY INHERITED FACTORS. 2075 01:32:34,680 --> 01:32:37,200 SO SOME SORT OF PREDISPOSITION 2076 01:32:37,200 --> 01:32:38,800 THAT YOU GET WHEN YOU'RE BORN 2077 01:32:38,800 --> 01:32:39,360 IN THE INTERACTION WITH THE 2078 01:32:39,360 --> 01:32:42,880 ENVIRONMENT I THINK ONE OF THE 2079 01:32:42,880 --> 01:32:44,440 QUESTIONS THAT CAME UP IN THE 2080 01:32:44,440 --> 01:32:45,040 QUESTION AND ANSWER BOX ASKED 2081 01:32:45,040 --> 01:32:48,160 ABOUT THE QUESTION AND ANSWER 2082 01:32:48,160 --> 01:32:49,720 AND TO THE GENES AND THE RISK 2083 01:32:49,720 --> 01:32:52,360 OF DEVELOPING THAT OVER THE 2084 01:32:52,360 --> 01:32:54,480 COURSE OF A LIFETIME. IT'S 2085 01:32:54,480 --> 01:32:55,920 EXACTLY THE QUESTION THAT 2086 01:32:55,920 --> 01:32:57,000 RESEARCHERS ARE TRYING TO 2087 01:32:57,000 --> 01:32:57,720 ANSWER THROUGH THIS 2088 01:32:57,720 --> 01:32:58,760 TRANSFORMATIVE AWARD AND THEY 2089 01:32:58,760 --> 01:33:01,520 HAVE DEVELOPED APOLOGETIC RISK 2090 01:33:01,520 --> 01:33:03,520 THIS POTENTIALLY COULD EXPLAIN 2091 01:33:03,520 --> 01:33:04,720 SOME OF THAT RISK AND THE 2092 01:33:04,720 --> 01:33:05,240 QUESTION NOW IS HOW IT 2093 01:33:05,240 --> 01:33:09,320 INTERACTS WITH THE 2094 01:33:09,320 --> 01:33:10,760 ENVIRONMENT. ONE BIG PROBLEM 2095 01:33:10,760 --> 01:33:12,400 IS THAT ALL OF HER INFORMATION 2096 01:33:12,400 --> 01:33:13,280 NOW IS RETROSPECTIVE. PEOPLE 2097 01:33:13,280 --> 01:33:13,960 WITH ALS AND WE TRIED TO GO 2098 01:33:13,960 --> 01:33:15,200 BACK IN TIME TO FIGURE OUT 2099 01:33:15,200 --> 01:33:20,040 WHAT THEY MAY BE EXPOSED TO. 2100 01:33:20,040 --> 01:33:21,200 ONE PRIORITY WILL BE TO OBTAIN 2101 01:33:21,200 --> 01:33:23,200 INFORMATION AND EVEN MATERIALS 2102 01:33:23,200 --> 01:33:25,760 FROM PEOPLE AND TO SEE WHAT 2103 01:33:25,760 --> 01:33:27,080 HAPPENS TO THEM OVER TIME. 2104 01:33:27,080 --> 01:33:27,640 THIS WILL TRY TO DETERMINE 2105 01:33:27,640 --> 01:33:28,200 WHAT ARE THE FACTORS THAT WE 2106 01:33:28,200 --> 01:33:31,160 CAN MEASURE LATER ON AND 2107 01:33:31,160 --> 01:33:31,760 DETERMINE THEIR PROBABILITY OF 2108 01:33:31,760 --> 01:33:34,920 HELPING ALS. I THINK THAT IS 2109 01:33:34,920 --> 01:33:38,000 AN AREA THAT CAN CHANGE A LOT 2110 01:33:38,000 --> 01:33:39,480 IN THE NEXT SEVERAL YEARS. IT 2111 01:33:39,480 --> 01:33:40,200 WILL HELP US MODEL THE DISEASE 2112 01:33:40,200 --> 01:33:43,120 WAS WE IDENTIFY THOSE FACTORS. 2113 01:33:43,120 --> 01:33:47,520 >>: THANK YOU. 2114 01:33:47,520 --> 01:33:49,120 >>: I COULD SIMPLY ADD 2115 01:33:49,120 --> 01:33:51,200 THAT I THINK THAT WE CAN 2116 01:33:51,200 --> 01:33:51,760 RESPOND TO PROGRESS OVER THE 2117 01:33:51,760 --> 01:33:53,920 LAST COUPLE OF YEARS HAVING 2118 01:33:53,920 --> 01:33:57,560 ACCESS TO DATA SETS. IT WAS 2119 01:33:57,560 --> 01:34:02,560 MENTIONED ABOUT WORK AND 2120 01:34:02,560 --> 01:34:07,120 SUPPORT FOR THE NY DC. THAT 2121 01:34:07,120 --> 01:34:08,360 CERTAINLY HAS FACILITATED, NOT 2122 01:34:08,360 --> 01:34:10,200 ONLY IDENTIFY DISEASE CAUSING 2123 01:34:10,200 --> 01:34:11,760 MUTATIONS BUT ALSO GENETIC 2124 01:34:11,760 --> 01:34:14,040 MODIFIERS AND MARKERS. 2125 01:34:14,040 --> 01:34:16,040 THERE'S SUCH EXAMPLES AND I 2126 01:34:16,040 --> 01:34:17,040 THINK HAVE VERY IMPORTANT 2127 01:34:17,040 --> 01:34:18,800 IMPLICATIONS FOR THE DISEASE 2128 01:34:18,800 --> 01:34:23,000 PROCESS. AT THE END OF THE 2129 01:34:23,000 --> 01:34:27,000 DAY IT'S JUST AN INCREDIBLY 2130 01:34:27,000 --> 01:34:27,640 COMPLEX SPORADIC ALS DISEASE. 2131 01:34:27,640 --> 01:34:31,400 THERE'S MANY UNDERLINING 2132 01:34:31,400 --> 01:34:33,720 PATHOLOGIES, T DP 43 FOR 2133 01:34:33,720 --> 01:34:34,920 EXAMPLE, YOUR POST WITH THE 2134 01:34:34,920 --> 01:34:36,360 LOSS OF FUNCTION AS WELL AS 2135 01:34:36,360 --> 01:34:37,720 THE TOXIC GAIN OF FUNCTION AND 2136 01:34:37,720 --> 01:34:38,920 THAT UNDERSTANDING THOSE 2137 01:34:38,920 --> 01:34:40,720 EVENTS AND THEN TRYING TO 2138 01:34:40,720 --> 01:34:42,880 MODEL IT. WHETHER IT'S AN ISP 2139 01:34:42,880 --> 01:34:45,760 CULTURES ARE LOW MOUSE MODELS. 2140 01:34:45,760 --> 01:34:46,960 THIS ALSO PRESENTS SOME 2141 01:34:46,960 --> 01:34:48,760 LIMITATIONS BECAUSE OF THE 2142 01:34:48,760 --> 01:34:51,080 FACT THAT THERE IS SPECIES 2143 01:34:51,080 --> 01:34:52,680 DIFFERENCES IN TERMS OF HOW 2144 01:34:52,680 --> 01:34:55,360 THESE BIOLOGICAL PROCESSES AND 2145 01:34:55,360 --> 01:34:56,800 EVENTS AND SPLICING OCCUR FOR 2146 01:34:56,800 --> 01:34:57,720 EXAMPLE. IT'S A COMBINATION 2147 01:34:57,720 --> 01:34:58,840 OF UNDERSTANDING THE 2148 01:34:58,840 --> 01:34:59,440 COMPLEXITY OF THE DISEASE AND 2149 01:34:59,440 --> 01:35:04,120 THEN DEVELOPING THE 2150 01:35:04,120 --> 01:35:04,800 APPROPRIATE TOOLS AND PUBLICLY 2151 01:35:04,800 --> 01:35:06,560 AVAILABLE DATA SETS FOR US TO 2152 01:35:06,560 --> 01:35:11,160 MODEL THE SYSTEMS BETTER. 2153 01:35:11,160 --> 01:35:15,560 >>: THANK YOU SO MUCH. 2154 01:35:15,560 --> 01:35:16,360 THE SECOND CLARITY IS TO THEN 2155 01:35:16,360 --> 01:35:18,360 FOCUS ON A BETTER 2156 01:35:18,360 --> 01:35:21,440 UNDERSTANDING OF WHAT THIS 2157 01:35:21,440 --> 01:35:22,680 MICROORGANISM THAT TENDS TO 2158 01:35:22,680 --> 01:35:23,160 ANALYZE THE CLINICAL 2159 01:35:23,160 --> 01:35:23,600 [INDISTINCT] AND IN 2160 01:35:23,600 --> 01:35:26,480 PARTICULAR, WHY SOME NEURONS 2161 01:35:26,480 --> 01:35:28,200 ARE DYING FOR PEOPLE THAT LIVE 2162 01:35:28,200 --> 01:35:31,320 WITH ALS IN THIS NEURONS HAVE 2163 01:35:31,320 --> 01:35:33,640 DIED. AND THAT HAPPENS 2164 01:35:33,640 --> 01:35:34,920 SOMETIMES WITH THE SAME 2165 01:35:34,920 --> 01:35:39,880 MUTATION FOR EXAMPLE. SO 2166 01:35:39,880 --> 01:35:41,280 MICHAEL YOU HAVE EXPERIENCE IN 2167 01:35:41,280 --> 01:35:41,840 BOTH OF THESE AND CAN MAYBE 2168 01:35:41,840 --> 01:35:45,560 DESCRIBE WHAT WE KNOW ABOUT 2169 01:35:45,560 --> 01:35:47,920 THIS AND MECHANISMS BETWEEN 2170 01:35:47,920 --> 01:35:49,520 THESE DISEASES? 2171 01:35:49,520 --> 01:35:50,400 >>: YES, THIS IS A REALLY 2172 01:35:50,400 --> 01:35:51,720 IMPORTANT TOPIC THAT THE 2173 01:35:51,720 --> 01:35:52,280 WORKING GROUP FELT THAT WE 2174 01:35:52,280 --> 01:35:57,040 SHOULD ADDRESS SYSTEMATICALLY. 2175 01:35:57,040 --> 01:35:58,200 SO AS BOB BROWN AND MARK VERY 2176 01:35:58,200 --> 01:36:02,040 NICELY STATED IN THEIR 2177 01:36:02,040 --> 01:36:03,840 INTRODUCTIONS, FTD AND ALS IS 2178 01:36:03,840 --> 01:36:05,720 A SPECTRUM OF BURROW DISORDERS 2179 01:36:05,720 --> 01:36:07,960 AND THE PATIENT CAN OF FTD, 2180 01:36:07,960 --> 01:36:08,560 ALS AND CIGNA BOTH TOGETHER, 2181 01:36:08,560 --> 01:36:11,920 IT CAN HAVE A ONE FOR THE 2182 01:36:11,920 --> 01:36:13,720 OTHER ONE AFTER THE OTHER WORK 2183 01:36:13,720 --> 01:36:15,440 TOGETHER. WE UNDERSTAND THAT 2184 01:36:15,440 --> 01:36:17,160 THESE DISORDERS ARE RELATED 2185 01:36:17,160 --> 01:36:18,520 BOTH CLINICALLY BUT ALSO 2186 01:36:18,520 --> 01:36:20,040 GENETICALLY WITH A LOT OF 2187 01:36:20,040 --> 01:36:21,600 OVERLAPS OF CAUSAL GENES AND 2188 01:36:21,600 --> 01:36:23,760 FAMILIES. UNFORTUNATELY SOME 2189 01:36:23,760 --> 01:36:26,360 GENES ONLY CAUSE ALS OR ONLY 2190 01:36:26,360 --> 01:36:28,800 COST FTD. IT IS A RELATED THE 2191 01:36:28,800 --> 01:36:30,760 AGGREGATION AND CIVILIZATION 2192 01:36:30,760 --> 01:36:33,600 OF THIS COMMONALITY ACROSS 2193 01:36:33,600 --> 01:36:35,800 MANY TYPES OF FTD ANIMALS ALL 2194 01:36:35,800 --> 01:36:38,240 OF ALS. WHAT WE REALLY DON'T 2195 01:36:38,240 --> 01:36:40,840 UNDERSTAND IS WHY SOME PEOPLE 2196 01:36:40,840 --> 01:36:43,080 DEVELOP FTD AND OTHER PEOPLE 2197 01:36:43,080 --> 01:36:43,720 DEVELOP ALS? AND SOMETIMES AS 2198 01:36:43,720 --> 01:36:48,160 MENTIONED, IN A SINGLE FAMILY. 2199 01:36:48,160 --> 01:36:52,440 THIS IS GOING TO TELL US A LOT 2200 01:36:52,440 --> 01:36:54,000 ABOUT WHETHER THE TERM IS 2201 01:36:54,000 --> 01:36:54,560 SELECTIVE VULNERABILITY AND 2202 01:36:54,560 --> 01:36:56,240 TWICE A CERTAIN TYPE OF NEURON 2203 01:36:56,240 --> 01:36:58,960 DEGENERATES IN FROM THE CORTEX 2204 01:36:58,960 --> 01:37:01,640 OR THE TEMP ORAL LOBE AND THAT 2205 01:37:01,640 --> 01:37:02,840 ANOTHER TYPE OF NEURON 2206 01:37:02,840 --> 01:37:03,440 DEGENERATES AND ALS ONTO THE 2207 01:37:03,440 --> 01:37:04,040 MOTOR CORTEX WERE OFF TO THE 2208 01:37:04,040 --> 01:37:08,560 SPINAL CORD. SO, WHAT IS 2209 01:37:08,560 --> 01:37:10,520 DRIVING THAT EVEN WITHIN A 2210 01:37:10,520 --> 01:37:12,640 SINGLE FAMILY? ARE THESE 2211 01:37:12,640 --> 01:37:14,160 ADDITIONAL GENETIC RISK 2212 01:37:14,160 --> 01:37:15,960 FACTORS ARE PROTECTED GENES? 2213 01:37:15,960 --> 01:37:16,720 ARE THESE ENVIRONMENTAL RISK 2214 01:37:16,720 --> 01:37:18,120 THAT SOMEBODY WITHIN THE 2215 01:37:18,120 --> 01:37:20,640 FAMILY HAS BEEN EXPOSED TO TO 2216 01:37:20,640 --> 01:37:22,160 MAKE THEM FORM FTD IN ADDITION 2217 01:37:22,160 --> 01:37:24,520 TO ALS? THESE ARE REALLY 2218 01:37:24,520 --> 01:37:26,360 UNKNOWNS IN ONE OF THE BIG 2219 01:37:26,360 --> 01:37:28,240 CHALLENGES IS THAT GENETICS 2220 01:37:28,240 --> 01:37:29,960 BENEFITS FROM LARGE SAMPLE 2221 01:37:29,960 --> 01:37:30,560 POPULATIONS. BUT THIS IS 2222 01:37:30,560 --> 01:37:32,960 RELATIVELY RARE DISORDER SO 2223 01:37:32,960 --> 01:37:34,880 WERE GOING TO HAVE TO REALLY 2224 01:37:34,880 --> 01:37:35,760 HARNESS THE POWER OF 2225 01:37:35,760 --> 01:37:37,600 COLLABORATIVE EFFORTS 2226 01:37:37,600 --> 01:37:39,240 WORLDWIDE TO START TO DEVELOP 2227 01:37:39,240 --> 01:37:40,880 DATABASES THAT ARE SHARED AND 2228 01:37:40,880 --> 01:37:43,040 MINED ACROSS TO RESEARCHERS 2229 01:37:43,040 --> 01:37:44,040 AND IDENTIFYING THESE 2230 01:37:44,040 --> 01:37:45,320 CONTRIBUTE FACTORS THAT CAUSE 2231 01:37:45,320 --> 01:37:47,840 ONE TO DEVELOP FTD, ALS, OR 2232 01:37:47,840 --> 01:37:51,920 BOTH. I THINK IT'S GOING TO 2233 01:37:51,920 --> 01:37:52,480 BE CRUCIAL IN HIS HYPOTHESIS 2234 01:37:52,480 --> 01:37:53,760 GENERATE APPROACH TO THEN COME 2235 01:37:53,760 --> 01:37:54,360 BACK TO BASIC SCIENCE AND THEN 2236 01:37:54,360 --> 01:37:58,080 TO BE MODELED AND WITH ANIMAL 2237 01:37:58,080 --> 01:37:59,360 MODELS DETERMINE IF THOSE 2238 01:37:59,360 --> 01:38:01,000 FACTORS ARE THERE THAT MIGHT 2239 01:38:01,000 --> 01:38:02,920 BE CAUSAL OR ACTUALLY HAVING 2240 01:38:02,920 --> 01:38:05,440 AN IMPACT ON THE TYPE OF 2241 01:38:05,440 --> 01:38:06,280 NEURON THAT IS VULNERABLE AND 2242 01:38:06,280 --> 01:38:06,760 POTENTIALLY THE TYPE OF 2243 01:38:06,760 --> 01:38:13,800 PATHOLOGY THAT DEVELOPS. 2244 01:38:13,800 --> 01:38:14,320 >>: THANK YOU. SO, 2245 01:38:14,320 --> 01:38:14,880 ANOTHER THING THAT WE HAVE 2246 01:38:14,880 --> 01:38:19,560 DISCUSSED A LOT AND WHY SOME 2247 01:38:19,560 --> 01:38:21,080 INDIVIDUALS WOULD BE RESISTANT 2248 01:38:21,080 --> 01:38:28,200 TO ALS? AS THEY ARE CARRYING 2249 01:38:28,200 --> 01:38:30,240 THAT MUTATION. AND RELATED TO 2250 01:38:30,240 --> 01:38:31,440 THIS EQUATION IS WHY IS THE 2251 01:38:31,440 --> 01:38:34,520 AGE OF ONSET OF ALS CAN BE SO 2252 01:38:34,520 --> 01:38:35,120 WIDELY DIFFERENT. SAMMY COULD 2253 01:38:35,120 --> 01:38:39,200 YOU PLEASE EXPLAIN SOME OF THE 2254 01:38:39,200 --> 01:38:40,320 DEFINING FACTORS UNDERLINING 2255 01:38:40,320 --> 01:38:40,880 THIS RESILIENCE AND WHY IT 2256 01:38:40,880 --> 01:38:42,360 MIGHT PROVIDE CRUCIAL 2257 01:38:42,360 --> 01:38:47,600 INFORMATION. 2258 01:38:47,600 --> 01:38:49,080 >>: I THINK YOU'RE RIGHT 2259 01:38:49,080 --> 01:38:50,360 THIS IS A REALLY IMPORTANT 2260 01:38:50,360 --> 01:38:51,960 QUESTION THAT I'M NOT SURE IF 2261 01:38:51,960 --> 01:38:52,560 WE REALLY PAY ENOUGH ATTENTION 2262 01:38:52,560 --> 01:38:58,160 TO A LOT OF TIMES. WERE VERY 2263 01:38:58,160 --> 01:38:59,760 FOCUSED ON THE CAUSES OF 2264 01:38:59,760 --> 01:39:00,800 DISEASE BUT WERE NOT ALWAYS 2265 01:39:00,800 --> 01:39:04,840 THINKING ABOUT WHAT TO PREVENT 2266 01:39:04,840 --> 01:39:06,280 DISEASE. WHILE THERE'S MANY 2267 01:39:06,280 --> 01:39:06,920 PEOPLE WHO GET ALS THERE'S 2268 01:39:06,920 --> 01:39:11,360 MANY PEOPLE WHO DO NOT. IS 2269 01:39:11,360 --> 01:39:12,120 THERE SOMETHING RATHER THAN 2270 01:39:12,120 --> 01:39:13,360 SOMETHING BEING PRESENT IN 2271 01:39:13,360 --> 01:39:15,760 PEOPLE WITH ALS OR IS IT 2272 01:39:15,760 --> 01:39:17,520 RATHER THAT THEY ARE MISSING A 2273 01:39:17,520 --> 01:39:20,600 FACTOR THAT'S IMPORTANT FOR 2274 01:39:20,600 --> 01:39:21,600 RESILIENCE? IT'S GOOD THAT WE 2275 01:39:21,600 --> 01:39:22,160 TALK ABOUT THIS NOW RIGHT 2276 01:39:22,160 --> 01:39:24,640 AFTER MICHAEL SPOKE ABOUT THE 2277 01:39:24,640 --> 01:39:26,840 FACT THAT SOME CELL TYPES ARE 2278 01:39:26,840 --> 01:39:28,360 SUSCEPTIBLE OR VULNERABLE ANTS 2279 01:39:28,360 --> 01:39:29,880 WERE THINKING ABOUT SOMETHING 2280 01:39:29,880 --> 01:39:31,920 THAT CAUSES DEAF IN THOSE 2281 01:39:31,920 --> 01:39:32,480 CELLS. BUT MAYBE THEY'RE 2282 01:39:32,480 --> 01:39:33,200 MISSING SOMETHING THAT MAKES 2283 01:39:33,200 --> 01:39:37,320 ALL OF THE REST OF THE SALES 2284 01:39:37,320 --> 01:39:39,880 RESISTANCE. IT'S THE WAY THAT 2285 01:39:39,880 --> 01:39:40,800 MANY OF OUR STUDIES ARE 2286 01:39:40,800 --> 01:39:42,640 CURRENTLY DESIGNED IS TO PICK 2287 01:39:42,640 --> 01:39:43,360 OUT THOSE RISK FACTORS BUT 2288 01:39:43,360 --> 01:39:44,160 THERE ARE METHODS AND 2289 01:39:44,160 --> 01:39:46,160 APPROACHES THAT WE CAN USE TO 2290 01:39:46,160 --> 01:39:46,840 LOOK AT THE OTHER SIDE OF THE 2291 01:39:46,840 --> 01:39:50,800 COIN TO FIND OUT IF THERE'S 2292 01:39:50,800 --> 01:39:52,760 OTHER FACTORS THAT PROMOTE 2293 01:39:52,760 --> 01:39:57,720 RESILIENCE THAT WE MAY NOT 2294 01:39:57,720 --> 01:39:59,040 HAVE NECESSARILY IDENTIFIED 2295 01:39:59,040 --> 01:40:00,840 BEFORE. THERE'S LOTS OF 2296 01:40:00,840 --> 01:40:02,040 LESSONS TO BE LEARNED HERE 2297 01:40:02,040 --> 01:40:04,200 FROM OTHER CONDITIONS AND 2298 01:40:04,200 --> 01:40:05,240 OTHER NEURODEGENERATIVE 2299 01:40:05,240 --> 01:40:06,520 DISORDERS, INCLUDING 2300 01:40:06,520 --> 01:40:07,960 ALZHEIMER'S DISEASE. THEY'VE 2301 01:40:07,960 --> 01:40:09,920 BEEN ABLE TO TAKE ADVANTAGE OF 2302 01:40:09,920 --> 01:40:11,800 POPULATIONS THAT HAVE GENETIC 2303 01:40:11,800 --> 01:40:13,080 PREDISPOSITIONS TO DISEASE AND 2304 01:40:13,080 --> 01:40:15,760 DIFFERENT ON SENSE OF AGES AND 2305 01:40:15,760 --> 01:40:17,440 FIND OUT WHAT FACTORS ARE 2306 01:40:17,440 --> 01:40:18,800 IMPORTANT FOR THE ONSET OF 2307 01:40:18,800 --> 01:40:20,160 AGING AND CAN FIND SOME SORT 2308 01:40:20,160 --> 01:40:23,600 OF FACTOR THAT DELAYS ONSET. 2309 01:40:23,600 --> 01:40:25,040 THIS IS OFTEN DONE IN GENETIC 2310 01:40:25,040 --> 01:40:27,400 STUDIES IN HUMANS BUT WITH 2311 01:40:27,400 --> 01:40:29,440 VERY LARGE ANIMAL STUDIES THAT 2312 01:40:29,440 --> 01:40:30,120 A SIMILAR APPROACH CAN BE 2313 01:40:30,120 --> 01:40:33,040 TAKEN TO IDENTIFY RESILIENCE 2314 01:40:33,040 --> 01:40:34,240 FACTORS. I THINK THERE'S A 2315 01:40:34,240 --> 01:40:34,840 LOT TO BE GAINED BY FOCUSING 2316 01:40:34,840 --> 01:40:41,480 ON THIS PARTICULAR ASPECT. 2317 01:40:41,480 --> 01:40:42,560 >>: THANK YOU. THE NEXT 2318 01:40:42,560 --> 01:40:44,280 THING I WOULD LIKE TO MENTION 2319 01:40:44,280 --> 01:40:45,880 AND I THINK IT'S ALREADY BEEN 2320 01:40:45,880 --> 01:40:46,520 INVOLVED BEFORE AND IT'S GREAT 2321 01:40:46,520 --> 01:40:49,360 TO COME BACK AND SIT DOWN AND 2322 01:40:49,360 --> 01:40:51,360 SEE THAT THERE IS A NEED TO 2323 01:40:51,360 --> 01:40:55,800 HAVE THIS AND TO UNDERSTAND 2324 01:40:55,800 --> 01:40:56,480 AND PREDICT WHAT OTHER EVENTS 2325 01:40:56,480 --> 01:40:58,320 CAN LEAD TO CONFESSION FROM 2326 01:40:58,320 --> 01:40:59,840 PRESENTING SYMPTOMATIC STAGES 2327 01:40:59,840 --> 01:41:05,000 OF ALS. WITH THE INDIVIDUAL 2328 01:41:05,000 --> 01:41:08,240 CARRIERS HEARING THIS 2329 01:41:08,240 --> 01:41:09,240 MUTATION. COOPER MAYBE YOU 2330 01:41:09,240 --> 01:41:09,880 CAN TELL US WHAT YOU THINK 2331 01:41:09,880 --> 01:41:13,440 ABOUT THIS AND THEN I THINK 2332 01:41:13,440 --> 01:41:15,120 THAT IF YOU CAN CONTRADICT 2333 01:41:15,120 --> 01:41:17,040 THAT DISCUSSION. 2334 01:41:17,040 --> 01:41:18,640 >>: AS YOU SAID I THINK 2335 01:41:18,640 --> 01:41:22,120 THIS IS GOING TO COME UP A 2336 01:41:22,120 --> 01:41:22,960 LOT. THIS IS CRITICALLY 2337 01:41:22,960 --> 01:41:23,440 IMPORTANT AND I THINK 2338 01:41:23,440 --> 01:41:26,480 UNDERSTUDIED TRADITIONALLY 2339 01:41:26,480 --> 01:41:28,000 ANYWAY. IN MY MIND THERE'S 2340 01:41:28,000 --> 01:41:30,200 REALLY TWO FUNDAMENTAL REASONS 2341 01:41:30,200 --> 01:41:30,840 WHY WE HAVE TO UNDERSTAND THIS 2342 01:41:30,840 --> 01:41:33,200 PROCESS. AS A MORAL AND THEN 2343 01:41:33,200 --> 01:41:34,400 THERE'S A PRACTICAL REASON. 2344 01:41:34,400 --> 01:41:35,120 MORALLY SPEAKING, AS A GENE 2345 01:41:35,120 --> 01:41:37,480 CARRIER DAYS FROM I HAVE NASTY 2346 01:41:37,480 --> 01:41:40,040 THE CIRCULATION TO MY ARM I 2347 01:41:40,040 --> 01:41:41,640 WANT TO BE ABLE TO READ 2348 01:41:41,640 --> 01:41:42,840 LITERATURE THAT CAN TELL ME 2349 01:41:42,840 --> 01:41:45,040 WHAT'S GOING ON AND WHAT I CAN 2350 01:41:45,040 --> 01:41:46,520 POTENTIALLY TAKE AND WHAT I 2351 01:41:46,520 --> 01:41:47,920 EXPECT FOR MY FUTURE. I THINK 2352 01:41:47,920 --> 01:41:50,760 A LOT OF FOLKS FEEL THAT WAY 2353 01:41:50,760 --> 01:41:51,320 TOO. AND THEN PRACTICALLY 2354 01:41:51,320 --> 01:41:54,320 SPEAKING, WE KNOW THIS DISEASE 2355 01:41:54,320 --> 01:41:56,000 THIS HAS BEEN STATED, IT 2356 01:41:56,000 --> 01:41:58,120 STARTS PROBABLY DECADES BEFORE 2357 01:41:58,120 --> 01:42:01,320 SYMPTOM ONSETS AND THE 2358 01:42:01,320 --> 01:42:01,880 COMPLEXITY OF THE BIOLOGICAL 2359 01:42:01,880 --> 01:42:03,760 PHENOMENON AT PLAY ONCE THE 2360 01:42:03,760 --> 01:42:05,680 DISEASE STARTS EXPANDS 2361 01:42:05,680 --> 01:42:07,760 EXPONENTIALLY. IT BECOMES 2362 01:42:07,760 --> 01:42:08,920 MUCH HARDER TO KNOW WHAT'S 2363 01:42:08,920 --> 01:42:12,160 GOING ON WE ARE LOOKING AT 2364 01:42:12,160 --> 01:42:14,240 INDIVIDUALS WHO PROBABLY HAVE 2365 01:42:14,240 --> 01:42:16,360 A LOT OF DIFFERENT 2366 01:42:16,360 --> 01:42:21,920 PATHOLOGIES. SO, IN ORDER TO 2367 01:42:21,920 --> 01:42:22,440 MEANINGFULLY DETER THIS 2368 01:42:22,440 --> 01:42:23,000 DISEASE AND TO MEANINGFULLY 2369 01:42:23,000 --> 01:42:26,040 UNDERSTAND WHAT'S GOING ON WE 2370 01:42:26,040 --> 01:42:27,160 NEED TO BEGIN SEARCHING MUCH 2371 01:42:27,160 --> 01:42:29,760 EARLIER. IN MOUSE MODELS, AND 2372 01:42:29,760 --> 01:42:36,920 I SEE DERIVED NEURAL LINES AND 2373 01:42:36,920 --> 01:42:40,000 YES. WITHIN THIS IS IMPORTANT 2374 01:42:40,000 --> 01:42:41,200 THAT WE INCLUDED THIS IN OUR 2375 01:42:41,200 --> 01:42:42,000 SUGGESTIONS AND THAT PEOPLE 2376 01:42:42,000 --> 01:42:44,280 BEGAN TO THINK IN A MORE 2377 01:42:44,280 --> 01:42:44,880 HOLISTIC TERM ABOUT WHEN THIS 2378 01:42:44,880 --> 01:42:50,280 DISEASE BEGINS AND ENDS. WE 2379 01:42:50,280 --> 01:42:53,320 MET I LIKE TO ADD HOW WE GO 2380 01:42:53,320 --> 01:42:57,160 ABOUT DOING THAT IS TO FOLLOW 2381 01:42:57,160 --> 01:42:58,120 INDIVIDUALS THAT HAVE DISEASE 2382 01:42:58,120 --> 01:42:58,920 CAUSING MUTATIONS SEE NINE FOR 2383 01:42:58,920 --> 01:43:00,720 EXAMPLE WOULD BE AN OBVIOUS 2384 01:43:00,720 --> 01:43:03,440 EXAMPLE OF WHERE YOU TRY TO 2385 01:43:03,440 --> 01:43:03,960 UNDERSTAND THE UNDERLYING 2386 01:43:03,960 --> 01:43:07,360 BIOLOGICAL MECHANISMS AND THEN 2387 01:43:07,360 --> 01:43:07,960 DEVELOP STRATEGIES. WHETHER 2388 01:43:07,960 --> 01:43:11,680 IT BE A BIOMARKER FOR A 2389 01:43:11,680 --> 01:43:13,800 BIOMARKER WOULD BE A CLEAR 2390 01:43:13,800 --> 01:43:17,800 EXAMPLE OF TRYING TO DETERMINE 2391 01:43:17,800 --> 01:43:18,200 WHETHER OR NOT IN 2392 01:43:18,200 --> 01:43:19,800 PRESYMPTOMATIC CARRIERS THERE 2393 01:43:19,800 --> 01:43:24,800 MIGHT BE AN INDICATION OF FINO 2394 01:43:24,800 --> 01:43:27,120 CONVERSION OR ESTATE OF 2395 01:43:27,120 --> 01:43:31,720 PROGNOSIS FOR THE INDIVIDUAL 2396 01:43:31,720 --> 01:43:36,640 WHO ONLY I BELIEVE, TO FEEL 2397 01:43:36,640 --> 01:43:37,240 THAT THAT TRANSLATES INTO 2398 01:43:37,240 --> 01:43:39,280 SPORADIC ALS AND THE WORK THAT 2399 01:43:39,280 --> 01:43:43,280 WE'RE DOING FROM A SPECIFIC 2400 01:43:43,280 --> 01:43:43,840 GENE CAUSING MUTATION WOULD 2401 01:43:43,840 --> 01:43:44,320 HAVE IMPLICATIONS FOR 2402 01:43:44,320 --> 01:43:47,160 OBVIOUSLY A MUCH LARGER GROUP 2403 01:43:47,160 --> 01:43:49,360 AND THIS WOULD BE A SPORADIC 2404 01:43:49,360 --> 01:43:51,240 DISEASE. I THINK MANY LABS 2405 01:43:51,240 --> 01:43:51,920 ARE PURSUING THAT AND I THINK 2406 01:43:51,920 --> 01:43:55,960 THAT I AM QUITE OPTIMISTIC 2407 01:43:55,960 --> 01:43:58,400 THAT IN THIS SPACE 2408 01:43:58,400 --> 01:44:00,240 UNDERSTANDING SPECIFIC DISEASE 2409 01:44:00,240 --> 01:44:01,880 CAUSING MUTATIONS IN THE 2410 01:44:01,880 --> 01:44:02,400 BROADER IMPLICATIONS FOR 2411 01:44:02,400 --> 01:44:03,040 SPORADIC ALS AND WITH WHAT 2412 01:44:03,040 --> 01:44:07,840 COOPER HAD JUST MENTIONED. 2413 01:44:07,840 --> 01:44:16,560 >>: THANK YOU. I THINK 2414 01:44:16,560 --> 01:44:17,160 THAT IS A VERY IMPORTANT ONE 2415 01:44:17,160 --> 01:44:19,200 WHICH IS REALLY HIGHLIGHTING 2416 01:44:19,200 --> 01:44:25,040 HOW WE MEET AND DEVELOP THESE 2417 01:44:25,040 --> 01:44:25,600 TECHNOLOGIES AND SUPPLIES 2418 01:44:25,600 --> 01:44:28,880 THESE TECHNOLOGIES TO UPDATE 2419 01:44:28,880 --> 01:44:32,800 MECHANISMS AND ALWAYS 2420 01:44:32,800 --> 01:44:33,840 QUANTIFIED IN THE SPECIFIC 2421 01:44:33,840 --> 01:44:34,440 TARGET. SO MICHAEL AND SAMMY 2422 01:44:34,440 --> 01:44:36,440 YOU CAN DESCRIBE WHITE IS THE 2423 01:44:36,440 --> 01:44:40,360 SO PARTICULAR IN ONE AND WITH 2424 01:44:40,360 --> 01:44:40,640 APOLOGIES? 2425 01:44:40,640 --> 01:44:47,440 >>: I THINK THAT A S ALS 2426 01:44:47,440 --> 01:44:48,320 FILLED HAS BEEN FANTASTIC AT 2427 01:44:48,320 --> 01:44:50,040 ABDUCTOR ANSWERS THE MISSED 2428 01:44:50,040 --> 01:44:52,880 OPPORTUNITIES OF THIS GROUP 2429 01:44:52,880 --> 01:44:54,680 HAS IDENTIFIED MY WIFE IS IN 2430 01:44:54,680 --> 01:44:56,960 THE ONCOLOGY ALWAYS REALIZE 2431 01:44:56,960 --> 01:44:58,360 THAT WE ARE ABOUT THE CANCER 2432 01:44:58,360 --> 01:45:02,840 FIELD AND TIME DURATIONS 2433 01:45:02,840 --> 01:45:03,600 CANCER AND A LOT OF WHAT MY 2434 01:45:03,600 --> 01:45:05,040 TEAM IS UNSUCCESSFUL HE HAS 2435 01:45:05,040 --> 01:45:05,760 COME FROM THE CANCER FIELD. 2436 01:45:05,760 --> 01:45:09,520 SOME OF THAT IT COMES FROM 2437 01:45:09,520 --> 01:45:10,000 SCREENING AND IDENTIFY 2438 01:45:10,000 --> 01:45:12,920 RESILIENCE FACTORS THAT SAMMY 2439 01:45:12,920 --> 01:45:14,640 MENTIONED THAT'S BEEN DONE BY 2440 01:45:14,640 --> 01:45:15,200 THE CANCER FIELD FOR A LONG 2441 01:45:15,200 --> 01:45:15,800 TIME AND WE NEED TO DO MORE OF 2442 01:45:15,800 --> 01:45:20,160 THAT HERE. SOME OF THOSE 2443 01:45:20,160 --> 01:45:20,760 OTHER USES FACIAL TRANSCRIPT 2444 01:45:20,760 --> 01:45:23,400 TONE EXCEPT MALL COLEY 2445 01:45:23,400 --> 01:45:25,320 MENTIONED. IT'S ONLY BEING 2446 01:45:25,320 --> 01:45:27,120 APPLIED ALS FIELD. SOMETHING 2447 01:45:27,120 --> 01:45:28,240 THAT WE DO WELL IN THIS FIELD 2448 01:45:28,240 --> 01:45:30,240 IS BEEN AN EARLY ADOPTION OF 2449 01:45:30,240 --> 01:45:33,280 HUMAN STEM CELL MODELS AND I 2450 01:45:33,280 --> 01:45:33,800 THINK THAT THERE'S MANY 2451 01:45:33,800 --> 01:45:34,360 INVESTIGATORS ON THIS PANEL 2452 01:45:34,360 --> 01:45:35,880 THAT USE THESE AND THINK ABOUT 2453 01:45:35,880 --> 01:45:39,560 THE FOREFRONT OF USING THESE 2454 01:45:39,560 --> 01:45:40,760 TWO MODEL FAMILIAL FORMS OF 2455 01:45:40,760 --> 01:45:44,640 THIS DISEASE AND THIS HAS NOW 2456 01:45:44,640 --> 01:45:47,000 GENERATED THE LARGEST 2457 01:45:47,000 --> 01:45:49,280 GENETICALLY LAUNCH ANY 2458 01:45:49,280 --> 01:45:50,920 DISEASE. I THINK IN SOME 2459 01:45:50,920 --> 01:45:51,400 RESPECTS WE ARE TAKING A 2460 01:45:51,400 --> 01:45:56,920 LEADERSHIP ROLE HERE. THIS 2461 01:45:56,920 --> 01:45:57,920 BEHOOVES US TO IDENTIFY OR 2462 01:45:57,920 --> 01:45:59,000 ADOPT SOME OF THESE NEW 2463 01:45:59,000 --> 01:46:00,640 TECHNOLOGIES THAT CAN BE 2464 01:46:00,640 --> 01:46:01,280 TRANSFORMATIVE IN OTHER FIELDS 2465 01:46:01,280 --> 01:46:03,120 AND THEN ALSO TO ENTICE SOME 2466 01:46:03,120 --> 01:46:04,560 OF THOSE INVESTIGATORS WHO ARE 2467 01:46:04,560 --> 01:46:06,240 LEADERS IN THEIR FIELDS TO 2468 01:46:06,240 --> 01:46:08,080 COME AND JOIN US IN THE ALS 2469 01:46:08,080 --> 01:46:10,040 FIELD AND WORK WITH US. WHO 2470 01:46:10,040 --> 01:46:10,720 MAYBE HAVE NEVER WORKED IN 2471 01:46:10,720 --> 01:46:11,680 THIS FIELD BEFORE BUT BRING A 2472 01:46:11,680 --> 01:46:17,120 LOT TO THE TABLE. 2473 01:46:17,120 --> 01:46:17,880 >>: I COMPLETELY AGREE 2474 01:46:17,880 --> 01:46:21,600 AND I KEEP THINKING ABOUT THIS 2475 01:46:21,600 --> 01:46:22,520 SAYING WHEN SOMEBODY TOLD ME 2476 01:46:22,520 --> 01:46:27,400 WHEN I FIRST STARTED AND OLD 2477 01:46:27,400 --> 01:46:31,200 QUESTIONS WITH NEW TECHNOLOGY 2478 01:46:31,200 --> 01:46:38,240 AND YOU CAN USE THAT THERE'S 2479 01:46:38,240 --> 01:46:38,840 SO MUCH NEW TECHNOLOGY OUT 2480 01:46:38,840 --> 01:46:41,480 THERE THAT MICHAEL ALLUDED TO 2481 01:46:41,480 --> 01:46:45,440 PEOPLE HAVE ALLUDED TO AND 2482 01:46:45,440 --> 01:46:47,280 HAVE PUT TOGETHER TO ANSWER 2483 01:46:47,280 --> 01:46:48,840 SPECIFIC QUESTIONS OUTSIDE THE 2484 01:46:48,840 --> 01:46:50,320 REALM OF ALS AND NEVERTHELESS 2485 01:46:50,320 --> 01:46:51,480 CAN STILL BE APPLIED TO THE 2486 01:46:51,480 --> 01:46:52,520 SAME QUESTIONS THAT WE HAVE 2487 01:46:52,520 --> 01:46:53,120 BEEN TRYING TO ANSWER FOR A 2488 01:46:53,120 --> 01:46:58,280 LONG TIME. ONE AREA THAT 2489 01:46:58,280 --> 01:47:08,880 COMES TO MIND IS TO THIS AND SMY 2490 01:47:41,200 --> 01:47:43,120 WITH SPATIAL TRANSFER SCHOOL 2491 01:47:43,120 --> 01:47:43,920 MAKES BUT IT GOES BEYOND THAT 2492 01:47:43,920 --> 01:47:49,720 TO LOOK BACK SPECIALIZED SORT 2493 01:47:49,720 --> 01:47:52,880 OF METHODS FOR CAPTURING 2494 01:47:52,880 --> 01:47:55,200 CHANGES TO DNA AND ART AND A 2495 01:47:55,200 --> 01:47:58,080 AND ONE OF THE STRUCTURE OF 2496 01:47:58,080 --> 01:48:00,240 RNA AND CHANGES THAT ARE 2497 01:48:00,240 --> 01:48:01,040 ANYTHING THAT AFFECTED DNA OR 2498 01:48:01,040 --> 01:48:04,360 RNA AND NOT AN ACTUAL CODE BUT 2499 01:48:04,360 --> 01:48:05,880 STILL CHANGES IN GENE 2500 01:48:05,880 --> 01:48:06,400 EXPRESSION DISPLACING AND 2501 01:48:06,400 --> 01:48:10,760 ACTIVITY IN TRANSLATION 2502 01:48:10,760 --> 01:48:13,600 TRANSCRIPTIONAL ACTIVITY IN 2503 01:48:13,600 --> 01:48:14,440 ANY NUMBER OF VERY SPECIFIC 2504 01:48:14,440 --> 01:48:17,000 APPROACHES THAT HAVE YET TO BE 2505 01:48:17,000 --> 01:48:22,200 APPLIED TO SOME OF THE MODELS 2506 01:48:22,200 --> 01:48:23,560 AND QUESTIONS ABOUT ALS. THIS 2507 01:48:23,560 --> 01:48:25,800 IS AN AREA THAT I AM SUPER 2508 01:48:25,800 --> 01:48:28,440 EXCITED ABOUT THIS CHANGES BY 2509 01:48:28,440 --> 01:48:30,720 THE MINUTE AND THAT IS PART OF 2510 01:48:30,720 --> 01:48:36,480 THE GOOD THING IN THAT CASE. 2511 01:48:36,480 --> 01:48:37,760 >>: THAT'S WHY THIS 2512 01:48:37,760 --> 01:48:39,120 CHANGES BY THE MINUTES AND 2513 01:48:39,120 --> 01:48:41,640 THAT IS BY IMPLEMENTING NEW 2514 01:48:41,640 --> 01:48:43,040 TECHNOLOGY IS VERY IMPORTANT 2515 01:48:43,040 --> 01:48:44,200 BECAUSE IT'S GOT CHALLENGES 2516 01:48:44,200 --> 01:48:45,200 INCLUDING, TO BE RECRUITS THIS 2517 01:48:45,200 --> 01:48:51,080 BUT ALSO TO RETRAIN THESE GENE 2518 01:48:51,080 --> 01:48:52,440 TRANSMITTALS AND MY LAST 2519 01:48:52,440 --> 01:48:55,600 QUESTION THIS DISCUSSION IS IF 2520 01:48:55,600 --> 01:48:58,840 YOU WANT TO ADDRESS THIS AND 2521 01:48:58,840 --> 01:49:06,560 HOW DO WE DO WITH THAT? 2522 01:49:06,560 --> 01:49:07,280 >>: I AM HAPPY TO DO SO. 2523 01:49:07,280 --> 01:49:10,560 FIRST I AM VERY HAPPY TO AGAIN 2524 01:49:10,560 --> 01:49:15,640 BE A PART OF THIS AND BE A 2525 01:49:15,640 --> 01:49:16,360 PART OF THIS ON THE CO- 2526 01:49:16,360 --> 01:49:17,640 PANELIST ENTERING YOUR PAPERS 2527 01:49:17,640 --> 01:49:18,600 AS I WAS A GRAD STUDENTS 2528 01:49:18,600 --> 01:49:20,080 ALREADY WERE WALKING THE WALK 2529 01:49:20,080 --> 01:49:20,880 WHEN WE SAY WERE TRAINING THE 2530 01:49:20,880 --> 01:49:23,200 NEXT GENERATION OF SCIENTISTS. 2531 01:49:23,200 --> 01:49:26,440 THANK YOU TO ALL FOR LAYING 2532 01:49:26,440 --> 01:49:28,480 THE GROUNDWORK FOR US NEW 2533 01:49:28,480 --> 01:49:33,000 SCIENTIST AS WELL. I THINK 2534 01:49:33,000 --> 01:49:35,320 WHAT I COULD DO IS WHEN WE SEE 2535 01:49:35,320 --> 01:49:35,880 SOMEBODY EXCITED TO JOIN THE 2536 01:49:35,880 --> 01:49:37,760 ALS WHO HAS A DIFFERENT 2537 01:49:37,760 --> 01:49:41,080 PERSPECTIVE IS TO INVITE THEM. 2538 01:49:41,080 --> 01:49:44,200 THIS CAN HAPPEN ACROSS ALL 2539 01:49:44,200 --> 01:49:45,800 FIELDS IS NOT UNIQUE TO ALS. 2540 01:49:45,800 --> 01:49:47,000 RESEARCH HAS CHANGED AND 2541 01:49:47,000 --> 01:49:48,400 PREPRINTS ARE OUT AND PEOPLE 2542 01:49:48,400 --> 01:49:48,960 ARE MORE COMFORTABLE SHARING 2543 01:49:48,960 --> 01:49:52,480 DATA AND WE ARE SEEING VERY 2544 01:49:52,480 --> 01:49:54,360 EXCITED PEOPLE THAT ARE COMING 2545 01:49:54,360 --> 01:49:58,520 IN FROM DIFFERENT FIELDS, LIKE 2546 01:49:58,520 --> 01:49:59,440 COMPUTER SCIENCE. AS MICHAEL 2547 01:49:59,440 --> 01:50:01,840 AND SAM WERE ALLUDING TO, THEY 2548 01:50:01,840 --> 01:50:02,920 ARE APPLYING NEW TECHNOLOGIES 2549 01:50:02,920 --> 01:50:03,480 AND NEW APPROACHES TO THE 2550 01:50:03,480 --> 01:50:06,240 FIELD AND THEY SHOULD BE 2551 01:50:06,240 --> 01:50:07,680 WELCOMED. I THINK WE SHOULD 2552 01:50:07,680 --> 01:50:09,640 ALL TRY TO BE THE BEST 2553 01:50:09,640 --> 01:50:10,680 VERSIONS OF OURSELVES AS WE 2554 01:50:10,680 --> 01:50:12,680 TRY TO MENTOR THE NEXT 2555 01:50:12,680 --> 01:50:13,760 GENERATION. MANY OF THESE 2556 01:50:13,760 --> 01:50:14,720 PEOPLE HERE ARE EXTREMELY 2557 01:50:14,720 --> 01:50:15,960 APPROACHABLE AND NICE TO WORK 2558 01:50:15,960 --> 01:50:16,720 WITH AND I'VE HAD THE 2559 01:50:16,720 --> 01:50:17,320 OPPORTUNITY TO WORK WITH THEM 2560 01:50:17,320 --> 01:50:24,960 AS WELL. I THINK WE CAN 2561 01:50:24,960 --> 01:50:27,120 CONTINUE TO DO THAT. WOULD WE 2562 01:50:27,120 --> 01:50:28,760 SEE A NEW SCIENTIST 2563 01:50:28,760 --> 01:50:29,400 APPROACHING US WITH NEW IDEAS, 2564 01:50:29,400 --> 01:50:32,000 LET'S SHARE DATA AND TO BE 2565 01:50:32,000 --> 01:50:34,760 OPEN. IF THEY HAVE NEW IDEAS 2566 01:50:34,760 --> 01:50:36,360 WE SHOULD WELCOME THEM AS 2567 01:50:36,360 --> 01:50:37,560 WELL. ACADEMIA AND THE WAY IT 2568 01:50:37,560 --> 01:50:39,400 IS SET UP, PUBLICATIONS ARE A 2569 01:50:39,400 --> 01:50:42,040 LITTLE BIT DIFFERENT. PEOPLE 2570 01:50:42,040 --> 01:50:43,320 CAN BE TERRITORIAL OF 2571 01:50:43,320 --> 01:50:44,440 AUTHORSHIP ET CETERA. BUT THE 2572 01:50:44,440 --> 01:50:46,160 MODEL IS CHANGING. I THINK 2573 01:50:46,160 --> 01:50:47,760 WERE NOW BEING MORE OPEN 2574 01:50:47,760 --> 01:50:50,320 BECAUSE THE DISEASES THAT WE 2575 01:50:50,320 --> 01:50:52,240 WORK WITH AFFECTED PATIENTS. 2576 01:50:52,240 --> 01:50:54,720 IT'S NOT JUST ABOUT OUR CAREER 2577 01:50:54,720 --> 01:50:55,280 GROWTH AS WELL. I THINK ALL 2578 01:50:55,280 --> 01:50:58,320 OF US AT DIFFERENT LEVELS IF 2579 01:50:58,320 --> 01:51:03,120 WE CAN ENCOURAGE NEW 2580 01:51:03,120 --> 01:51:04,760 SCIENTISTS. THE OLDER 2581 01:51:04,760 --> 01:51:06,840 SCIENTIST PERHAPS TO JUST 2582 01:51:06,840 --> 01:51:07,560 SHARE DATA OPENLY AND APPLYING 2583 01:51:07,560 --> 01:51:09,400 NEW METHODS AND AGAIN, BE THE 2584 01:51:09,400 --> 01:51:12,880 BEST VERSION OF OURSELVES AS 2585 01:51:12,880 --> 01:51:13,920 WE TRAINED GENERATION OF 2586 01:51:13,920 --> 01:51:14,480 SCIENTISTS I THINK WE COULD 2587 01:51:14,480 --> 01:51:18,800 TACKLE THIS DISEASE AND WE 2588 01:51:18,800 --> 01:51:19,480 COULD BE GOOD STEWARDESS AND 2589 01:51:19,480 --> 01:51:22,720 GOOD SCIENTISTS AS WELL. 2590 01:51:22,720 --> 01:51:23,480 EMILY YOU CERTAINLY HAVE A LOT 2591 01:51:23,480 --> 01:51:27,120 MORE EXPERIENCE THAN I DO SO I 2592 01:51:27,120 --> 01:51:30,520 WELCOME YOUR COMMENTS. 2593 01:51:30,520 --> 01:51:31,080 >>: THERE'S A WELCOMING 2594 01:51:31,080 --> 01:51:33,000 NEW PEOPLE INTO THE FIELDS AND 2595 01:51:33,000 --> 01:51:34,760 THERE'S ACTIVELY GOING OUT 2596 01:51:34,760 --> 01:51:35,440 INTO TESTING THEIR ARMS AND 2597 01:51:35,440 --> 01:51:36,040 STRONGLY URGING THEM TO CHANGE 2598 01:51:36,040 --> 01:51:42,640 THE GROUP AND THEN 2599 01:51:42,640 --> 01:51:44,360 FACILITATING THE ADOPTION OF 2600 01:51:44,360 --> 01:51:45,000 NEW TECHNOLOGIES REQUIRES 2601 01:51:45,000 --> 01:51:46,800 TRAINING AND DISSEMINATION. 2602 01:51:46,800 --> 01:51:48,880 SOMETIMES IT REQUIRES HANDS ON 2603 01:51:48,880 --> 01:51:50,480 TRAINING AND WORKSHOPS AND 2604 01:51:50,480 --> 01:51:50,960 SOMETIMES IT REQUIRES 2605 01:51:50,960 --> 01:51:52,640 DIFFERENT INCENTIVES FOR 2606 01:51:52,640 --> 01:51:53,360 EXPERTS, SUCH AS EXPERTS IN 2607 01:51:53,360 --> 01:51:55,520 THOSE METHODS TO JOIN THE 2608 01:51:55,520 --> 01:51:56,000 FIELDS AND ALL OF THESE 2609 01:51:56,000 --> 01:52:00,680 EFFORTS ARE UNDERWAY. CAN SEE 2610 01:52:00,680 --> 01:52:02,560 IT IN THE CHANGES IN FUNDING 2611 01:52:02,560 --> 01:52:04,080 OPPORTUNITY ANNOUNCEMENTS FROM 2612 01:52:04,080 --> 01:52:07,120 THE NIH, FROM PUBLIC AND 2613 01:52:07,120 --> 01:52:08,080 PRIVATE PARTNERSHIPS. THE 2614 01:52:08,080 --> 01:52:09,200 FOCUSES MORE ON COLLABORATION 2615 01:52:09,200 --> 01:52:10,400 AND ON BRINGING IN NEW 2616 01:52:10,400 --> 01:52:11,040 TECHNOLOGIES TO THE FIELD AND 2617 01:52:11,040 --> 01:52:16,360 INCENTIVIZING THIS THROUGH AN 2618 01:52:16,360 --> 01:52:17,040 EMPHASIS ON INTERDISCIPLINARY 2619 01:52:17,040 --> 01:52:21,440 TEAM SCIENCE AND ON THE 2620 01:52:21,440 --> 01:52:22,200 DEVELOPMENT OF COURSES AND 2621 01:52:22,200 --> 01:52:23,480 BOOKSHOPS THAT TEACH PEOPLE 2622 01:52:23,480 --> 01:52:24,000 HOW TO DO THIS AND ON 2623 01:52:24,000 --> 01:52:26,920 PUBLISHING VIDEOS THAT SHOW 2624 01:52:26,920 --> 01:52:30,760 PEOPLE IMPLEMENTING THESE 2625 01:52:30,760 --> 01:52:32,000 METHODS. SO, IT'S NOT GOING 2626 01:52:32,000 --> 01:52:33,200 TO BE ONE SOLUTION. IT'S 2627 01:52:33,200 --> 01:52:34,960 GOING TO BE A COMBINATION OF 2628 01:52:34,960 --> 01:52:40,040 SOLUTIONS TAILORED TO HAVE THE 2629 01:52:40,040 --> 01:52:40,640 MOST STRATEGICALLY TAILORED TO 2630 01:52:40,640 --> 01:52:46,480 HAVE THE MOST IMPACT. 2631 01:52:46,480 --> 01:52:48,640 >>: THANK YOU SAMMY AND 2632 01:52:48,640 --> 01:52:49,680 EVERYONE. THIS IS THE END OF 2633 01:52:49,680 --> 01:52:52,080 THIS DISCUSSION AND WE ARE NOW 2634 01:52:52,080 --> 01:52:53,800 GOING TO SPEND OUR REMAINING 2635 01:52:53,800 --> 01:52:56,200 TIME TO ANSWER AS MANY 2636 01:52:56,200 --> 01:52:56,760 QUESTIONS AS POSSIBLE ABOUT 2637 01:52:56,760 --> 01:52:57,320 THE PRIORITIES IDENTIFIED IN 2638 01:52:57,320 --> 01:53:02,200 THIS WORKING GROUP. FOR 2639 01:53:02,200 --> 01:53:04,000 EVERYONE FEEL FREE TO CONTINUE 2640 01:53:04,000 --> 01:53:05,600 TO ENTER QUESTIONS USING THE 2641 01:53:05,600 --> 01:53:06,200 ZOOM QUEUE AND A BOX. ALL OF 2642 01:53:06,200 --> 01:53:08,760 THE QUESTION AND COMMENTS WILL 2643 01:53:08,760 --> 01:53:12,480 BE ON THE WEBSITE. WE MAY NOT 2644 01:53:12,480 --> 01:53:13,400 HAVE TIME TO ANSWER ALL 2645 01:53:13,400 --> 01:53:13,880 QUESTIONS THAT WE ARE 2646 01:53:13,880 --> 01:53:16,360 COLLECTING THEM AND WE WILL 2647 01:53:16,360 --> 01:53:19,560 CONSIDER THEM. WE MAY COMBINE 2648 01:53:19,560 --> 01:53:22,480 SOME QUESTIONS AND WE WILL 2649 01:53:22,480 --> 01:53:24,440 SYNTHESIZE QUESTIONS AND 2650 01:53:24,440 --> 01:53:27,480 ANSWERS ON THE MEETING 2651 01:53:27,480 --> 01:53:29,040 WEBSITES AFTER THIS EVENT. WE 2652 01:53:29,040 --> 01:53:29,640 HAVE RECEIVED A FEW QUESTIONS 2653 01:53:29,640 --> 01:53:34,720 AND WE WILL READ THEM AND 2654 01:53:34,720 --> 01:53:35,960 MEMBERS OF THE PANEL WILL 2655 01:53:35,960 --> 01:53:36,560 ANSWER THEM AS MANY QUESTIONS 2656 01:53:36,560 --> 01:53:43,160 AS WE CAN. YOU WANT TO START? 2657 01:53:43,160 --> 01:53:44,240 >>: THANK YOU. BUT I AM 2658 01:53:44,240 --> 01:53:45,960 AFRAID THAT YOU GET THEM BOTH 2659 01:53:45,960 --> 01:53:48,600 TO QUESTIONS, BOTH OF YOU. 2660 01:53:48,600 --> 01:53:50,640 THE FIRST ONE IS, HOW ARE WE 2661 01:53:50,640 --> 01:53:51,960 MAKING FORMS OF ALS AND 2662 01:53:51,960 --> 01:53:54,120 GENETIC CAUSES OF ALS THAT 2663 01:53:54,120 --> 01:53:56,960 OCCUR IN LOW AND MIDDLE INCOME 2664 01:53:56,960 --> 01:53:58,480 COUNTRIES A PRIORITY? QUITE 2665 01:53:58,480 --> 01:53:59,760 OBVIOUSLY BIOLOGY AND MENTAL 2666 01:53:59,760 --> 01:54:00,960 FACTORS ARE SIGNIFICANTLY 2667 01:54:00,960 --> 01:54:02,680 DIFFERENT IN THESE COUNTRIES 2668 01:54:02,680 --> 01:54:04,040 AND FOR THESE POPULATIONS 2669 01:54:04,040 --> 01:54:05,000 COMPARED TO THE GLOBAL NORTH. 2670 01:54:05,000 --> 01:54:06,760 EQUITY DICTATES IT WOULD MAKE 2671 01:54:06,760 --> 01:54:08,720 A CONCERTED EFFORT TO INCLUDE 2672 01:54:08,720 --> 01:54:10,120 RESEARCH IS RELEVANT FOR THESE 2673 01:54:10,120 --> 01:54:10,840 POPULATIONS. COVER THIS A 2674 01:54:10,840 --> 01:54:14,680 LITTLE BIT WOULD YOU LIKE TO 2675 01:54:14,680 --> 01:54:14,960 START? 2676 01:54:14,960 --> 01:54:15,560 >>: I DID COVER AND I DO 2677 01:54:15,560 --> 01:54:18,800 WANT TO RECOGNIZE THE EFFORTS 2678 01:54:18,800 --> 01:54:20,800 WHO ARE WORKING HARD ON THIS 2679 01:54:20,800 --> 01:54:21,920 AS WELL. SOMETIME THE CALLS 2680 01:54:21,920 --> 01:54:23,960 ARE AT 6:00 A.M. SO I MISS 2681 01:54:23,960 --> 01:54:25,680 THEM BUT HE IS GRACIOUS ENOUGH 2682 01:54:25,680 --> 01:54:27,080 TO SEND EVERYBODY THE MINUTE 2683 01:54:27,080 --> 01:54:29,280 SO THAT'S WONDERFUL. WHAT WE 2684 01:54:29,280 --> 01:54:30,920 NEED OUR SAMPLE SIZES FROM 2685 01:54:30,920 --> 01:54:33,560 THESE COUNTRIES. WE NEED TO 2686 01:54:33,560 --> 01:54:35,560 SUPPORT OBTAINING GRANTS, 2687 01:54:35,560 --> 01:54:36,800 WEATHERS NIH GRANTS ARE LOCAL 2688 01:54:36,800 --> 01:54:39,120 GRANTS AS WELL. I KNOW THIS 2689 01:54:39,120 --> 01:54:40,120 DISCUSSIONS AT THESE MEETINGS 2690 01:54:40,120 --> 01:54:41,080 ABOUT LOCAL GRANTS THAT GROUPS 2691 01:54:41,080 --> 01:54:44,280 CAN GET AS WELL. SO WHAT ARE 2692 01:54:44,280 --> 01:54:44,800 THE END OF SENDING DATA TO 2693 01:54:44,800 --> 01:54:48,160 GROUPS THAT ARE SET UP TO 2694 01:54:48,160 --> 01:54:49,800 PROCESS DATA, IT WOULD BE 2695 01:54:49,800 --> 01:54:51,520 PREFERABLE FOR THEM TO HAVE 2696 01:54:51,520 --> 01:54:52,680 FULL AUTONOMY OVER THEIR DATA. 2697 01:54:52,680 --> 01:54:53,240 THEY ARE THE ONES ANALYZING 2698 01:54:53,240 --> 01:54:56,520 THEIR DATA. THERE IS AN 2699 01:54:56,520 --> 01:54:57,120 EFFORT AND THERE NEEDS TO BE A 2700 01:54:57,120 --> 01:55:01,000 BIGGER EFFORT AND WE DO SEE 2701 01:55:01,000 --> 01:55:03,240 THAT IN THE PSYCHIATRIC GENOME 2702 01:55:03,240 --> 01:55:04,600 CONSORTIUM GROUP. ALSO VERY 2703 01:55:04,600 --> 01:55:05,240 LUCKY TO HAVE AN AMAZING 2704 01:55:05,240 --> 01:55:07,120 STATISTICAL GENETICIST IN 2705 01:55:07,120 --> 01:55:08,280 AUSTRALIA, NAOMI RAY OF 2706 01:55:08,280 --> 01:55:11,600 COURSE. I WENT TO REQUEST ALL 2707 01:55:11,600 --> 01:55:14,000 OF HER BECAUSE SHE DOES A LOT 2708 01:55:14,000 --> 01:55:14,840 OF WORK FOR OUTSIDE OF THE 2709 01:55:14,840 --> 01:55:18,320 PRIMARY EUROPEAN COHORT AS 2710 01:55:18,320 --> 01:55:20,640 WELL. BUT WE NEED ARE SAMPLE 2711 01:55:20,640 --> 01:55:22,320 SIZES. I THINK DOCTOR MICHAEL 2712 01:55:22,320 --> 01:55:24,360 WARD ALSO ILLUSTRATED THIS AS 2713 01:55:24,360 --> 01:55:26,200 WELL. WE ALL HAVE A DIFFERENT 2714 01:55:26,200 --> 01:55:29,200 GENETIC RISK, IS THE GENETIC 2715 01:55:29,200 --> 01:55:29,920 RISK FROM A DIFFERENT 2716 01:55:29,920 --> 01:55:32,360 POPULATION PROTECTIVE? OR IS 2717 01:55:32,360 --> 01:55:34,560 IT ACTUALLY COMBINED WITH 2718 01:55:34,560 --> 01:55:40,560 SEPARATE SYSTEM? SO, THE 2719 01:55:40,560 --> 01:55:41,800 EFFORTS ARE THERE AND THEY 2720 01:55:41,800 --> 01:55:44,000 NEED TO BE AMPLIFIED AND 2721 01:55:44,000 --> 01:55:45,480 AGAIN, THIS WAS ONE OF THE 2722 01:55:45,480 --> 01:55:46,960 MAIN PRIORITIES LISTED BY OUR 2723 01:55:46,960 --> 01:55:50,040 GROUP. LET'S SEE HOW THINGS 2724 01:55:50,040 --> 01:55:53,120 GO IN THE FUTURE. BUT SAMPLE 2725 01:55:53,120 --> 01:55:53,720 SIZES FROM THESE GEOGRAPHICAL 2726 01:55:53,720 --> 01:55:55,800 AREAS THAT ARE MISSED IN THE 2727 01:55:55,800 --> 01:55:56,360 CURRENT STUDIES IS WHAT IS 2728 01:55:56,360 --> 01:55:59,520 ABSOLUTELY NEEDED TODAY. 2729 01:55:59,520 --> 01:56:04,720 >>: THANK YOU. 2730 01:56:04,720 --> 01:56:05,640 >>: SOMETIMES WE ARE 2731 01:56:05,640 --> 01:56:06,560 LIMITED BY RESEARCH 2732 01:56:06,560 --> 01:56:07,440 INFRASTRUCTURE IN THESE 2733 01:56:07,440 --> 01:56:08,240 LOCATIONS AND THAT'S WHY 2734 01:56:08,240 --> 01:56:08,800 COLLECTING LARGE NUMBERS CAN 2735 01:56:08,800 --> 01:56:13,800 BE CHALLENGING. BUT AS A 2736 01:56:13,800 --> 01:56:14,480 MULTI, MODEL DATA SETS GROW 2737 01:56:14,480 --> 01:56:19,000 AND AS WE LEARN HOW SOME 2738 01:56:19,000 --> 01:56:21,840 ASPECTS OF WHAT YOU CAN STUDY 2739 01:56:21,840 --> 01:56:22,720 ADDITIONALLY TO GENOTYPE AND 2740 01:56:22,720 --> 01:56:23,280 ENVIRONMENTS, PERHAPS WE CAN 2741 01:56:23,280 --> 01:56:27,960 BRIDGE SOME OF THESE GAPS BY 2742 01:56:27,960 --> 01:56:29,360 STUDYING SAMPLES THAT ARE 2743 01:56:29,360 --> 01:56:31,120 RELATIVELY MORE FEASIBLE TO 2744 01:56:31,120 --> 01:56:33,440 CORRECT ON THESE POPULATIONS 2745 01:56:33,440 --> 01:56:35,400 AND LINKING STUDIES IN THOSE 2746 01:56:35,400 --> 01:56:36,560 TYPES OF SAMPLES AND LEARNING 2747 01:56:36,560 --> 01:56:37,160 FROM THE LARGER DATA SETS THAT 2748 01:56:37,160 --> 01:56:40,680 WE HAVE ALREADY COLLECTED. 2749 01:56:40,680 --> 01:56:43,080 THAT ALSO EMPHASIZES THE 2750 01:56:43,080 --> 01:56:46,080 IMPORTANCE OF MAXIMIZING 2751 01:56:46,080 --> 01:56:47,040 INFORMATION THAT YOU CAN 2752 01:56:47,040 --> 01:56:47,880 COLLECT FROM COHORTS THAT IT 2753 01:56:47,880 --> 01:56:48,440 IS POSSIBLE FOR YOU TO STUDY 2754 01:56:48,440 --> 01:56:53,680 AT SCALE. ASPECTS OF THESE 2755 01:56:53,680 --> 01:56:54,160 CAN INFORM MORE FOCUSED 2756 01:56:54,160 --> 01:57:00,840 STUDIES WHERE FEASIBLE. 2757 01:57:00,840 --> 01:57:01,640 >>: INFRASTRUCTURE KEY IS 2758 01:57:01,640 --> 01:57:03,680 THAT HE IS IN IT AND BEING 2759 01:57:03,680 --> 01:57:06,240 ACTIVE IS VERY IMPORTANT. BUT 2760 01:57:06,240 --> 01:57:08,200 MAKING SURE THAT WE DO ENGAGE 2761 01:57:08,200 --> 01:57:10,560 THE RESEARCHERS IN LOCAL 2762 01:57:10,560 --> 01:57:11,040 COMMUNITIES. THE SECOND 2763 01:57:11,040 --> 01:57:14,520 QUESTION IS AGAIN FOR BOTH OF 2764 01:57:14,520 --> 01:57:15,760 YOU, GEOCODE INCLUDE PEOPLE 2765 01:57:15,760 --> 01:57:16,680 WITH C9 EXPANSION IN YOUR 2766 01:57:16,680 --> 01:57:18,080 STUDIES? I AM IN FRANCE AND 2767 01:57:18,080 --> 01:57:22,200 WE ARE MANY AND ALL OVER THE 2768 01:57:22,200 --> 01:57:22,760 WORLD. HOW ABOUT YOU START 2769 01:57:22,760 --> 01:57:22,920 US. 2770 01:57:22,920 --> 01:57:26,000 >>: WE ABSOLUTELY DO AND 2771 01:57:26,000 --> 01:57:30,200 IF THEY ARE IN FRANCE, THEN 2772 01:57:30,200 --> 01:57:32,320 THEY ARE ALREADY PART OF A 2773 01:57:32,320 --> 01:57:34,640 PROJECT OF MINE. THIS IS THE 2774 01:57:34,640 --> 01:57:35,520 LARGEST AIRLESS GENOME 2775 01:57:35,520 --> 01:57:35,960 SEQUENCING STUDY AND 2776 01:57:35,960 --> 01:57:42,240 MULTIMODAL CITY IN THE WORLD. 2777 01:57:42,240 --> 01:57:44,000 WE MAKE A CONCERTED EFFORT TO 2778 01:57:44,000 --> 01:57:45,280 INCLUDE EVERYBODY HE WANTS TO 2779 01:57:45,280 --> 01:57:46,280 PARTICIPATE, THAT'S THE WHOLE 2780 01:57:46,280 --> 01:57:46,720 POINT OF HIS LARGE, 2781 01:57:46,720 --> 01:57:50,720 MULTINATIONAL STUDIES. 2782 01:57:50,720 --> 01:57:52,760 >>: I JUST WANT TO 2783 01:57:52,760 --> 01:57:54,000 CLARIFY, BECAUSE I UNDERSTAND 2784 01:57:54,000 --> 01:57:55,760 WHY THIS QUESTION EVEN AROSE. 2785 01:57:55,760 --> 01:57:58,040 IT ALSO DEPENDS ON YOUR 2786 01:57:58,040 --> 01:57:59,160 RESEARCH QUESTION. FOR 2787 01:57:59,160 --> 01:57:59,880 EXAMPLE, AND TRADITIONAL 2788 01:57:59,880 --> 01:58:00,760 ASSOCIATION STUDIES PEOPLE 2789 01:58:00,760 --> 01:58:04,120 WANT TO FIND A NEW GENETIC 2790 01:58:04,120 --> 01:58:04,800 SIGNATURE. A LOT OF THE TIME 2791 01:58:04,800 --> 01:58:08,760 ACTUALLY INDIVIDUALS THAT ARE 2792 01:58:08,760 --> 01:58:11,200 POSITIVE FOR KNOWN CAUSAL 2793 01:58:11,200 --> 01:58:13,160 MUTATIONS, LIKE C9 ARE 72 ARE 2794 01:58:13,160 --> 01:58:16,840 EXCLUDED IN THAT THEY MAY OR 2795 01:58:16,840 --> 01:58:18,440 THEY ARE GOING TO GIVE A 2796 01:58:18,440 --> 01:58:19,200 SIGNAL OF SOMETHING WE ALREADY 2797 01:58:19,200 --> 01:58:25,480 KNOW, THAT BEING SAID DESPITE 2798 01:58:25,480 --> 01:58:26,880 EFFORTS TO EXCLUDE AND THEN 2799 01:58:26,880 --> 01:58:29,000 FINDING SOMETHING NOVEL, THE 2800 01:58:29,000 --> 01:58:31,280 TOP HIT IS ALWAYS SOD ONE. 2801 01:58:31,280 --> 01:58:32,320 THAT MEANS THAT WERE DOING THE 2802 01:58:32,320 --> 01:58:33,120 SIGNS CORRECTLY. IT'S NOT 2803 01:58:33,120 --> 01:58:36,680 THAT WE ARE EXCLUDING THEM TO 2804 01:58:36,680 --> 01:58:37,160 EXCLUDE, IT FOLLOWS THE 2805 01:58:37,160 --> 01:58:39,680 RESEARCH QUESTION. 2806 01:58:39,680 --> 01:58:42,600 ALTERNATIVELY, I THINK IT WAS 2807 01:58:42,600 --> 01:58:43,160 BOTH TWO OF OUR PANELISTS 2808 01:58:43,160 --> 01:58:45,800 MENTIONED THIS AS WELL. THERE 2809 01:58:45,800 --> 01:58:49,560 IS A FAMILIAR WORLD BUILDING 2810 01:58:49,560 --> 01:58:51,360 AS WELL AS INTERNAL 2811 01:58:51,360 --> 01:58:52,600 VARIABILITY AND C9 IS A 2812 01:58:52,600 --> 01:58:55,000 CLASSIC EXAMPLE OF THAT AS 2813 01:58:55,000 --> 01:58:55,880 WELL. SOME INDIVIDUALS 2814 01:58:55,880 --> 01:58:57,640 PRESENT WITH ALS, SOME WITH 2815 01:58:57,640 --> 01:58:59,200 FTD, AND SOME COMBINED. IS 2816 01:58:59,200 --> 01:58:59,760 THERE ANOTHER GENETIC HIT OR 2817 01:58:59,760 --> 01:59:04,840 IS OR ANYTHING ELSE HAPPENING. 2818 01:59:04,840 --> 01:59:05,520 THAT QUESTION SPECIFICALLY 2819 01:59:05,520 --> 01:59:07,760 TARGETS INDIVIDUALS AND 2820 01:59:07,760 --> 01:59:08,840 INCLUDE INDIVIDUALS WITH SEAT 2821 01:59:08,840 --> 01:59:10,680 NINE OR SEVEN TO EXPANSION. 2822 01:59:10,680 --> 01:59:11,840 IT DEPENDS ON THE RESEARCH 2823 01:59:11,840 --> 01:59:12,680 QUESTION AS WELL. BUT OF 2824 01:59:12,680 --> 01:59:16,160 COURSE, EVERYBODY IS WELCOME 2825 01:59:16,160 --> 01:59:18,640 AS WAS JUST MENTIONED. 2826 01:59:18,640 --> 01:59:19,640 >>: ACTUALLY SOME 2827 01:59:19,640 --> 01:59:20,600 QUESTIONS THAT YOU ARE 2828 01:59:20,600 --> 01:59:21,760 MENTIONED, SOME QUESTIONS CAN 2829 01:59:21,760 --> 01:59:22,760 ONLY BE ANSWERED BY TAKING A 2830 01:59:22,760 --> 01:59:28,760 PEOPLE ONLY WITH C9 OR OTHER 2831 01:59:28,760 --> 01:59:29,840 EXPANSION. 2832 01:59:29,840 --> 01:59:30,600 >>: WOULD BE GRATEFUL IF 2833 01:59:30,600 --> 01:59:32,600 YOU COULD TRY TO ANSWER A 2834 01:59:32,600 --> 01:59:33,320 DIFFICULT BUT IMPORTANT 2835 01:59:33,320 --> 01:59:35,480 QUESTION. WHICH IS, WHAT IS 2836 01:59:35,480 --> 01:59:37,600 THE BEST EFFORT FOR 2837 01:59:37,600 --> 01:59:38,200 IDENTIFYING AND VALIDATING NEW 2838 01:59:38,200 --> 01:59:41,480 SURVEY TARGETS? 2839 01:59:41,480 --> 01:59:42,360 >>: I THINK THIS IS 2840 01:59:42,360 --> 01:59:44,360 REALLY CHALLENGING BECAUSE 2841 01:59:44,360 --> 01:59:45,560 IT'S INCREDIBLY EXPANSIVE. 2842 01:59:45,560 --> 01:59:46,400 MAYBE SOME EXAMPLES WOULD 2843 01:59:46,400 --> 01:59:48,280 HELP. IN MY OPINION MOST OF 2844 01:59:48,280 --> 01:59:50,760 OUR PROMISING TARGETS HAVE 2845 01:59:50,760 --> 01:59:53,840 COME EITHER FROM GENETICS OR 2846 01:59:53,840 --> 01:59:55,800 FROM HUMAN PATHOLOGY FROM 2847 01:59:55,800 --> 01:59:56,520 PEOPLE WHO UNFORTUNATELY DIED 2848 01:59:56,520 --> 01:59:59,440 OF ALS. A GREAT EXAMPLE IS 2849 01:59:59,440 --> 02:00:01,400 SOD ONE DISCOVERED BY BOB 2850 02:00:01,400 --> 02:00:03,080 BROWN WHICH EXPERIENCED 2851 02:00:03,080 --> 02:00:04,600 SEVERAL DECADES AND AFTER MUCH 2852 02:00:04,600 --> 02:00:06,240 RESEARCH INTO THE MECHANISMS 2853 02:00:06,240 --> 02:00:07,720 OF HOW THOSE MUTATIONS CAN'T 2854 02:00:07,720 --> 02:00:09,040 NEURON DEAF, WE NOW HAVE A 2855 02:00:09,040 --> 02:00:11,800 VERY PROMISING PHASE THREE 2856 02:00:11,800 --> 02:00:17,040 THERAPY THAT WAS DEVELOPED. 2857 02:00:17,040 --> 02:00:19,160 AND THEN OF COURSE, FOR NON- 2858 02:00:19,160 --> 02:00:21,920 FAMILIAL CAUSES, THE DISCOVERY 2859 02:00:21,920 --> 02:00:22,920 OF TB 343 GLOBALIZATION WAS 2860 02:00:22,920 --> 02:00:25,720 FOUND IN 97% OF PATIENTS WITH 2861 02:00:25,720 --> 02:00:31,360 ALS. THIS REALLY PROVIDES A 2862 02:00:31,360 --> 02:00:31,880 MECHANISM INTO STUDYING 2863 02:00:31,880 --> 02:00:33,680 SPORADIC ALS FOR PATIENTS WE 2864 02:00:33,680 --> 02:00:37,640 DON'T HAVE FAMILIAL GENES TO 2865 02:00:37,640 --> 02:00:39,120 ASSOCIATE WITH THIS DISEASE. 2866 02:00:39,120 --> 02:00:41,560 IN MUCH WORK SINCE DISCOVERED 2867 02:00:41,560 --> 02:00:43,560 IN 2006 HAS LED TO AN 2868 02:00:43,560 --> 02:00:44,760 UNDERSTANDING OF MECHANISMS OF 2869 02:00:44,760 --> 02:00:45,880 ACTION. WHEN HE GETS LOST 2870 02:00:45,880 --> 02:00:47,120 FROM A NUCLEAR IS THAT BAD 2871 02:00:47,120 --> 02:00:50,480 THINGS HAPPEN THE SPLICING OF 2872 02:00:50,480 --> 02:00:52,160 GENES AND ONE OF THE MAJOR 2873 02:00:52,160 --> 02:00:52,960 DISCOVERERS OF SOME OF THE 2874 02:00:52,960 --> 02:00:54,600 GENES THAT BECOME SPLICE CAN 2875 02:00:54,600 --> 02:00:55,120 BE SEEN IN BOTH CELLULAR 2876 02:00:55,120 --> 02:00:59,640 MODELS AND PATIENTS WITH 2877 02:00:59,640 --> 02:01:01,240 DISEASE. AND NOW THERE'S A 2878 02:01:01,240 --> 02:01:02,800 NUMBER OF DRUG COMPANIES TO 2879 02:01:02,800 --> 02:01:04,880 DEVELOP EFFORTS TO TARGET THAT 2880 02:01:04,880 --> 02:01:06,240 SPLICING BIOLOGY THAT IS GOING 2881 02:01:06,240 --> 02:01:08,280 ON. I THINK THOSE HAVE BEEN 2882 02:01:08,280 --> 02:01:09,680 EARLY LEADS FROM STRONG GENES 2883 02:01:09,680 --> 02:01:11,680 THAT CAUSE FAMILIAR FORMS OF 2884 02:01:11,680 --> 02:01:13,080 DISEASE AND PATHOLOGY THAT'S 2885 02:01:13,080 --> 02:01:14,400 OBVIOUS. THE NEXT CHALLENGES 2886 02:01:14,400 --> 02:01:17,120 ARE GOING TO BE TO IDENTIFY 2887 02:01:17,120 --> 02:01:19,160 THOSE OTHER CAUSES OF SPORADIC 2888 02:01:19,160 --> 02:01:19,920 PROVIDERS AND WE'VE BEEN 2889 02:01:19,920 --> 02:01:20,440 DISCUSSING THIS DURING THE 2890 02:01:20,440 --> 02:01:23,720 SESSION AND HOW TO MODEL THOSE 2891 02:01:23,720 --> 02:01:26,760 IN CELLULAR OR ANIMAL MODELS. 2892 02:01:26,760 --> 02:01:27,240 FIGURE OUT INTERVENING 2893 02:01:27,240 --> 02:01:28,640 PATHWAYS IF THEY IMPROVE THOSE 2894 02:01:28,640 --> 02:01:29,200 MODELS AND THEN ULTIMATELY 2895 02:01:29,200 --> 02:01:32,480 WORKING WITH COMPANIES TO 2896 02:01:32,480 --> 02:01:33,040 DEVELOP THERAPIES AND THEN 2897 02:01:33,040 --> 02:01:38,400 TESTING THOSE HYPOTHESES IN 2898 02:01:38,400 --> 02:01:41,440 TRIALS. 2899 02:01:41,440 --> 02:01:42,000 >>: I THINK AN IMPORTANT 2900 02:01:42,000 --> 02:01:45,800 POINT IS WE TALKED ABOUT 2901 02:01:45,800 --> 02:01:46,480 QUANTITY EMERGING TECHNOLOGIES 2902 02:01:46,480 --> 02:01:48,480 AND I FELT LIKE I DID IN THE 2903 02:01:48,480 --> 02:01:50,840 PAST MAYBE TEN-YEARS AGO OR 2904 02:01:50,840 --> 02:01:52,040 SO. WE WERE WORKING UNDER 2905 02:01:52,040 --> 02:01:53,920 SOME ASSUMPTIONS. BUT IF YOU 2906 02:01:53,920 --> 02:01:54,480 CAN LOOK AT ADVANCES IN CRYO 2907 02:01:54,480 --> 02:01:58,960 EM TECHNOLOGIES IN MASS -BASED 2908 02:01:58,960 --> 02:02:00,640 APPROACHES, THIS APPROACH IS 2909 02:02:00,640 --> 02:02:02,760 ARE NOW TURNING US INTO MUCH 2910 02:02:02,760 --> 02:02:04,880 SMARTER SCIENTIST BECAUSE FOR 2911 02:02:04,880 --> 02:02:05,720 UNDERSTANDING THE STRUCTURES 2912 02:02:05,720 --> 02:02:06,200 OF HIS DISEASE-RELATED 2913 02:02:06,200 --> 02:02:07,400 PROTEINS WHICH NORMALLY 2914 02:02:07,400 --> 02:02:10,560 ACCUMULATE. THE POTENTIAL 2915 02:02:10,560 --> 02:02:11,120 PROTEIN POST- TRANSLATIONAL 2916 02:02:11,120 --> 02:02:11,600 MODIFICATIONS THAT ARE 2917 02:02:11,600 --> 02:02:15,240 ASSOCIATED FOR EXAMPLE WITH T 2918 02:02:15,240 --> 02:02:15,800 DP FOUR THREE, HOW DOES 2919 02:02:15,800 --> 02:02:17,680 PHOSPHOR RELATIONAL OR 2920 02:02:17,680 --> 02:02:18,720 SEDATION INFLUENCE THAT 2921 02:02:18,720 --> 02:02:21,080 SHUTTLING BETWEEN THE NUCLEAR 2922 02:02:21,080 --> 02:02:24,920 AND CYTOPLASMIC COMPARTMENTS? 2923 02:02:24,920 --> 02:02:25,480 THAT WAS CERTAINLY A THING 2924 02:02:25,480 --> 02:02:27,280 THAT WAS ECHOED WITHIN THIS 2925 02:02:27,280 --> 02:02:31,240 WORK GROUP IS HOW EMERGING 2926 02:02:31,240 --> 02:02:36,000 TECHNOLOGIES ARE IMPACTING, 2927 02:02:36,000 --> 02:02:37,520 AND ONLY DNA AND RNA 2928 02:02:37,520 --> 02:02:38,080 CAPABILITIES, BUT CERTAINLY 2929 02:02:38,080 --> 02:02:46,840 PROTEIN STRUCTURES AS WELL. 2930 02:02:46,840 --> 02:02:47,600 >>: DO YOU WANT TO ASK 2931 02:02:47,600 --> 02:02:47,920 THE NEXT ONE? 2932 02:02:47,920 --> 02:02:50,880 >>: THANK YOU. THIS 2933 02:02:50,880 --> 02:02:59,120 QUESTION IS GOING TO PEER AND 2934 02:02:59,120 --> 02:03:00,840 SCOOPER. OUR LARGE FAMILY OF 2935 02:03:00,840 --> 02:03:03,040 FAMILIAL ALS AND DEVASTATED AS 2936 02:03:03,040 --> 02:03:04,680 WE CONTINUALLY LOSE FAMILY 2937 02:03:04,680 --> 02:03:05,440 MEMBERS FROM THIS HORRIFIC 2938 02:03:05,440 --> 02:03:06,640 DISEASE. THE ALS GENE HAS 2939 02:03:06,640 --> 02:03:08,360 BEEN IDENTIFIED BUT IS NOT AS 2940 02:03:08,360 --> 02:03:10,560 COMMON AS SOME OF THE OTHER 2941 02:03:10,560 --> 02:03:11,280 ALS GENES. OUR QUESTION TO 2942 02:03:11,280 --> 02:03:14,840 YOU IS WHAT IT ALL MAKES SENSE 2943 02:03:14,840 --> 02:03:16,360 TO START INCREASING GENE 2944 02:03:16,360 --> 02:03:16,960 CARRIERS ON MEDICATION AS SOON 2945 02:03:16,960 --> 02:03:18,200 AS POSSIBLE? THIS MAY ALLOW 2946 02:03:18,200 --> 02:03:19,560 FOR A SLOWER PROGRESSION OF 2947 02:03:19,560 --> 02:03:21,880 THE DISEASE AND MAY EVEN HOLD 2948 02:03:21,880 --> 02:03:22,840 THE DISEASE. OUR FAMILY HAS 2949 02:03:22,840 --> 02:03:24,680 SADLY WITNESSED TOO MANY 2950 02:03:24,680 --> 02:03:26,160 MEMBERS WHO BARELY LAST WHEN 2951 02:03:26,160 --> 02:03:27,640 YOU'RE WITH THIS DISEASE. 2952 02:03:27,640 --> 02:03:28,760 SINCE HER SUIT FEW SUCH 2953 02:03:28,760 --> 02:03:31,080 OPTIONS IT WOULD MAKE NO SENSE 2954 02:03:31,080 --> 02:03:31,880 TO STOP THESE APPRECIATIVE 2955 02:03:31,880 --> 02:03:32,560 OCCASIONS TO IMPROVE THESE 2956 02:03:32,560 --> 02:03:33,360 GENE CARRIERS. AS A 2957 02:03:33,360 --> 02:03:34,680 GRANDMOTHER WITH A HUSBAND TO 2958 02:03:34,680 --> 02:03:38,120 HAVE THE DISEASE I'VE SEEN MY 2959 02:03:38,120 --> 02:03:38,720 CHILDREN AND MY GRANDCHILDREN 2960 02:03:38,720 --> 02:03:39,840 PASS FROM ALS, EACH AND EVERY 2961 02:03:39,840 --> 02:03:41,040 DAY OF MY LIFE I'M REMINDED OF 2962 02:03:41,040 --> 02:03:42,480 THE LOVE ONCE I HAVE LOST AND 2963 02:03:42,480 --> 02:03:43,480 I WILL CONTINUALLY FIGHT FOR 2964 02:03:43,480 --> 02:03:45,560 THE TREATMENT OF THE DISEASE 2965 02:03:45,560 --> 02:03:50,760 UNTIL MY DYING DAY. COOPER 2966 02:03:50,760 --> 02:03:53,280 PERHAPS YOU WOULD LIKE TO 2967 02:03:53,280 --> 02:03:53,480 START. 2968 02:03:53,480 --> 02:03:57,520 >>: MAYBE I CAN START 2969 02:03:57,520 --> 02:04:04,280 COOPER. I WENT TO THINK YOU 2970 02:04:04,280 --> 02:04:04,840 FOR THIS IMPORTANT QUESTION 2971 02:04:04,840 --> 02:04:06,880 AND IT'S A VERY MOVING 2972 02:04:06,880 --> 02:04:10,480 QUESTION. I WILL SAY THAT WE 2973 02:04:10,480 --> 02:04:11,160 ARE ORGANIZE AND TO RECOGNIZE 2974 02:04:11,160 --> 02:04:12,400 HOW IMPORTANT IT IS TO TAKE 2975 02:04:12,400 --> 02:04:14,480 PATIENTS EARLY AND AGAIN, I 2976 02:04:14,480 --> 02:04:16,480 WILL SAY BETWEEN THEORY AND 2977 02:04:16,480 --> 02:04:17,080 PRINCIPLE YES. IT WOULD MAKE 2978 02:04:17,080 --> 02:04:18,800 SENSE TO START TREATMENT EARLY 2979 02:04:18,800 --> 02:04:22,480 ON AND REGARDLESS OF SYMPTOM. 2980 02:04:22,480 --> 02:04:24,120 BUT I THINK IN REALITY THE 2981 02:04:24,120 --> 02:04:24,760 SITUATION IS A LITTLE BIT MORE 2982 02:04:24,760 --> 02:04:27,800 COMPLICATED THAN THAT BECAUSE 2983 02:04:27,800 --> 02:04:30,400 EACH OF THE DRUGS THAT ARE 2984 02:04:30,400 --> 02:04:31,400 APPROVED THAT HAVE POTENTIAL 2985 02:04:31,400 --> 02:04:37,080 SIDE EFFECTS AND WE KNOW WHAT 2986 02:04:37,080 --> 02:04:37,680 THE POTENTIAL SIDE EFFECTS OF 2987 02:04:37,680 --> 02:04:39,480 LENO AND THEREFORE, WHEN WE 2988 02:04:39,480 --> 02:04:42,920 ARE DOING THIS AND AS A 2989 02:04:42,920 --> 02:04:45,040 PHYSICIAN IN THE ABSENCE OF 2990 02:04:45,040 --> 02:04:46,400 DISEASE IT IS REALLY HARD TO 2991 02:04:46,400 --> 02:04:46,960 JUSTIFY GIVING THE PATIENT A 2992 02:04:46,960 --> 02:04:52,600 DRUG THAT HAS OR MAY HAVE 2993 02:04:52,600 --> 02:04:53,200 SERIOUS SIDE EFFECTS. NOT TO 2994 02:04:53,200 --> 02:04:57,600 MENTION THE FACT, IN TODAY'S 2995 02:04:57,600 --> 02:04:58,160 DAY GENETIC COUNSELORS TELL 2996 02:04:58,160 --> 02:05:01,640 PACERS AND TELL US THAT EVEN 2997 02:05:01,640 --> 02:05:07,200 THE FAMILIAL PATIENT THAT GENE 2998 02:05:07,200 --> 02:05:08,200 CARRIERS MAY NOT NECESSARILY 2999 02:05:08,200 --> 02:05:12,600 DEVELOP THIS DISEASE. I WOULD 3000 02:05:12,600 --> 02:05:13,920 LIKE TO REASSURE HER THAT 3001 02:05:13,920 --> 02:05:14,520 AGAIN, AS WE DISCUSSED EARLIER 3002 02:05:14,520 --> 02:05:17,920 TODAY THAT EFFORTS THAT WE ARE 3003 02:05:17,920 --> 02:05:18,480 UNDERGOING NOW AND EVERYBODY 3004 02:05:18,480 --> 02:05:20,040 UNDERSTANDS THE IMPORTANCE 3005 02:05:20,040 --> 02:05:23,200 DIAGNOSED EARLY AND TO START 3006 02:05:23,200 --> 02:05:23,720 TREATMENT EARLY, EVEN UP 3007 02:05:23,720 --> 02:05:25,600 RECENT EMETIC PHASES. THERE 3008 02:05:25,600 --> 02:05:28,000 ARE STUDIES UNDERGOING NOW AND 3009 02:05:28,000 --> 02:05:34,760 A DOCTOR IN MIAMI FLORIDA FOR 3010 02:05:34,760 --> 02:05:35,720 INSTANCE IN DOCTORS AT 3011 02:05:35,720 --> 02:05:36,320 MASSACHUSETTS GENERAL HOSPITAL 3012 02:05:36,320 --> 02:05:37,760 REALLY FOLLOW REFLECTIVE 3013 02:05:37,760 --> 02:05:39,240 CARRIERS AND WIDE VARIETY OF 3014 02:05:39,240 --> 02:05:43,280 GENE MUTATION KNOWN TO CAUSE 3015 02:05:43,280 --> 02:05:47,160 ALS. THIS IS REALLY A 3016 02:05:47,160 --> 02:05:47,920 LONGITUDINAL AND NATIONAL 3017 02:05:47,920 --> 02:05:48,480 BIOMARKER PEOPLE WHO ARE 3018 02:05:48,480 --> 02:05:53,800 FAMILY MEMBERS THAT WE KNOW 3019 02:05:53,800 --> 02:05:56,520 ARE DEVELOPING ALS AND 3020 02:05:56,520 --> 02:05:57,280 RECOGNIZING THAT ALS SYMPTOMS 3021 02:05:57,280 --> 02:05:59,000 CAN BEGIN AT ANY AGE. THE 3022 02:05:59,000 --> 02:05:59,680 CRITICAL OBSTACLES THAT WE DO 3023 02:05:59,680 --> 02:06:03,480 NOT HAVE A BIOMARKER. IN 3024 02:06:03,480 --> 02:06:05,720 TERMS OF THAT STUDY I THINK 3025 02:06:05,720 --> 02:06:08,360 THAT BY LOOKING AT SYMPTOMATIC 3026 02:06:08,360 --> 02:06:08,840 PATIENTS CARING FOR GENE 3027 02:06:08,840 --> 02:06:10,640 MUTATION RESEARCHERS HAVE 3028 02:06:10,640 --> 02:06:14,480 FOUND FILAMENT LEVELS AND THEY 3029 02:06:14,480 --> 02:06:16,800 RISE IN THE SIX-12 MONTHS 3030 02:06:16,800 --> 02:06:20,000 PRIOR TO THE CLINICAL DISEASE. 3031 02:06:20,000 --> 02:06:21,680 NOW THIS NARROW FILAMENT I 3032 02:06:21,680 --> 02:06:25,400 BELIEVE HAS KEY BECAUSE WE MAY 3033 02:06:25,400 --> 02:06:31,480 NOW BE ABLE TO USE INCREASING 3034 02:06:31,480 --> 02:06:32,720 BLOOD FILAMENT LEVELS TO 3035 02:06:32,720 --> 02:06:33,800 IDENTIFIED INDIVIDUALS WITH 3036 02:06:33,800 --> 02:06:36,320 ANY GENE MUTATIONS, AND EVEN 3037 02:06:36,320 --> 02:06:37,600 PERHAPS CONVERT THIS TRANSFER 3038 02:06:37,600 --> 02:06:39,120 WITH SPORADIC PATIENTS WHO ARE 3039 02:06:39,120 --> 02:06:39,680 THE GREATEST SHORT-TERM RISK 3040 02:06:39,680 --> 02:06:43,840 OF DEVELOPING ALS. THEREFORE, 3041 02:06:43,840 --> 02:06:45,480 WE MAY ENROLL AN INCREASING 3042 02:06:45,480 --> 02:06:46,120 AMOUNT OF TRIALS AND EVEN 3043 02:06:46,120 --> 02:06:46,760 START TREATMENT WITH THE 3044 02:06:46,760 --> 02:06:50,040 APPROVED DRUGS. THE GOALS FOR 3045 02:06:50,040 --> 02:06:54,720 THIS BIOMARKER HAVE BEEN 3046 02:06:54,720 --> 02:06:55,280 SYMPTOMATIC TRIALS HAVE LED 3047 02:06:55,280 --> 02:06:58,800 THE FOUNDATION FOR DELAYING 3048 02:06:58,800 --> 02:06:59,320 THE ONSET PERHAPS IN 3049 02:06:59,320 --> 02:06:59,840 PREVENTING THE DISEASE BY 3050 02:06:59,840 --> 02:07:03,640 STARTING TRIALS EARLY AND BY 3051 02:07:03,640 --> 02:07:05,560 STARTING DRUGS OF THE 3052 02:07:05,560 --> 02:07:06,400 PRESYMPTOMATIC WERE SMALL 3053 02:07:06,400 --> 02:07:10,800 SYMPTOMATIC LEVELS. I'M SURE, 3054 02:07:10,800 --> 02:07:13,240 CLARITY ITSELF I THINK THAT 3055 02:07:13,240 --> 02:07:16,680 COOPER WILL HAVE A MEANINGFUL 3056 02:07:16,680 --> 02:07:23,560 ANSWER TO THIS QUESTION. 3057 02:07:23,560 --> 02:07:24,920 >>: THIS IS A VERY 3058 02:07:24,920 --> 02:07:25,520 IMPORTANT QUESTION LIKE THAT 3059 02:07:25,520 --> 02:07:27,320 WAS SET AT THE BEGINNING. I 3060 02:07:27,320 --> 02:07:29,480 APPRECIATE YOU BEING SO 3061 02:07:29,480 --> 02:07:30,720 VULNERABLE IN SHARING YOUR 3062 02:07:30,720 --> 02:07:32,240 FAMILY STORY. I HAVE A 3063 02:07:32,240 --> 02:07:33,560 SIMILAR SITUATION AND IS VERY 3064 02:07:33,560 --> 02:07:34,920 HEARTBREAKING AND SCARY. YOU 3065 02:07:34,920 --> 02:07:35,640 WANT TO ASK, I FEEL THAT WAY 3066 02:07:35,640 --> 02:07:39,560 ALL OF THE TIME. YOU WANT TO 3067 02:07:39,560 --> 02:07:42,760 ACT, TAKE SOMETHING, AND DO 3068 02:07:42,760 --> 02:07:43,680 SOMETHING. THERE'S NOT A 3069 02:07:43,680 --> 02:07:45,160 REALLY GREAT TO REASON, IN MY 3070 02:07:45,160 --> 02:07:46,920 OPINION FOR THIS. AS IT WAS 3071 02:07:46,920 --> 02:07:49,560 SAID A LOT OF IT COMES DOWN TO 3072 02:07:49,560 --> 02:07:50,240 THAT PREVIOUSLY WE DIDN'T HAVE 3073 02:07:50,240 --> 02:07:50,840 GREAT BIOMARKER SINCE IT WAS 3074 02:07:50,840 --> 02:07:53,480 REALLY HARD TO RUN THESE 3075 02:07:53,480 --> 02:07:57,840 STUDIES. HIS [INDISTINCT] 3076 02:07:57,840 --> 02:07:58,840 THERE IS VARIABLE PENETRANCE 3077 02:07:58,840 --> 02:08:00,520 WHICH IS TO SAY WHICH WAS 3078 02:08:00,520 --> 02:08:01,760 STATED BEFORE THAT THIS 3079 02:08:01,760 --> 02:08:03,120 DISEASE SHOWS UP IN DIFFERENT 3080 02:08:03,120 --> 02:08:04,280 WAYS AT DIFFERENT TIMES AND IT 3081 02:08:04,280 --> 02:08:04,880 BECOMES REALLY CHALLENGING TO 3082 02:08:04,880 --> 02:08:09,120 RUN A STUDY. NOW WE HAVE 3083 02:08:09,120 --> 02:08:10,760 BETTER TOOLS AND PEOPLE 3084 02:08:10,760 --> 02:08:12,400 RECOGNIZE THIS IS AN IMPORTANT 3085 02:08:12,400 --> 02:08:13,560 UNDERTAKING AND THEY ARE DOING 3086 02:08:13,560 --> 02:08:15,240 IT. I HOPE WE HAVE SOME 3087 02:08:15,240 --> 02:08:16,160 RESULTS FOR YOU SOON AND AS I 3088 02:08:16,160 --> 02:08:19,880 SAID PREVIOUSLY, I THINK THIS 3089 02:08:19,880 --> 02:08:23,120 IS A MORAL ISSUE, WE SHOULD BE 3090 02:08:23,120 --> 02:08:24,680 TAKING DRUGS AND COMPANIES AND 3091 02:08:24,680 --> 02:08:25,840 RESEARCHERS ARE RECOGNIZING IN 3092 02:08:25,840 --> 02:08:26,600 THE FUNCTIONAL ISSUE TOO. AT 3093 02:08:26,600 --> 02:08:28,200 THE END OF THE DAY YOU MAY NOT 3094 02:08:28,200 --> 02:08:28,720 GET GOOD RESULTS IF YOU'RE 3095 02:08:28,720 --> 02:08:32,200 WAITING TOO LONG. IT MAY BE 3096 02:08:32,200 --> 02:08:34,000 REALLY IMPORTANT TO DETER 3097 02:08:34,000 --> 02:08:34,960 DISEASE AT EARLY STAGES, JUST 3098 02:08:34,960 --> 02:08:38,440 SCIENTIFICALLY WE NEEDED TO 3099 02:08:38,440 --> 02:08:39,520 UNDERSTANDS. THANK YOU FOR 3100 02:08:39,520 --> 02:08:40,800 YOUR QUESTION. 3101 02:08:40,800 --> 02:08:41,360 >>: THANK YOU COOPER. 3102 02:08:41,360 --> 02:08:42,880 I'M GOING TO TAKE ONE OF THE 3103 02:08:42,880 --> 02:08:43,960 COMMENTS OF THE CHAT AS WELL 3104 02:08:43,960 --> 02:08:46,480 AND HIGHLIGHTED AS WELL. SO 3105 02:08:46,480 --> 02:08:48,640 JEAN WHO ALSO SITS ON ONE OF 3106 02:08:48,640 --> 02:08:50,000 THE PANELS HAS SAID, AVERAGE 3107 02:08:50,000 --> 02:08:53,600 LIFE EXPECTANCY OF A SEAT 90 3108 02:08:53,600 --> 02:08:54,760 CARRIERS 65, ACCOUNTING FOR 3109 02:08:54,760 --> 02:08:55,480 THE 90% PENETRANCE. 3110 02:08:55,480 --> 02:08:56,040 HIV-POSITIVE PEOPLE WERE PUT 3111 02:08:56,040 --> 02:08:58,680 ON DRUGS WITH NOTE WITH SIDE 3112 02:08:58,680 --> 02:08:59,400 EFFECTS OF THOSE SIGNS EVIDENT 3113 02:08:59,400 --> 02:09:01,600 DISEASE ACTIVITY TO GREAT 3114 02:09:01,600 --> 02:09:05,240 EFFECT. I SUPPOSE AT THAT 3115 02:09:05,240 --> 02:09:06,720 TIME THERE MIGHT'VE BEEN A 3116 02:09:06,720 --> 02:09:09,120 BIOMARKER OF HIV INFECTION OF 3117 02:09:09,120 --> 02:09:09,680 COURSE, BUT NECESSARILY A 3118 02:09:09,680 --> 02:09:10,240 BIOMARKER OF TRANSFORMATION 3119 02:09:10,240 --> 02:09:17,480 INTO AIDS. 3120 02:09:17,480 --> 02:09:18,000 >>: NEXT QUESTION FOR 3121 02:09:18,000 --> 02:09:21,840 SAMMY AND IT IS ASKED IN A 3122 02:09:21,840 --> 02:09:26,440 PROGRESSIVE USING THE REPEAT 3123 02:09:26,440 --> 02:09:29,240 [INDISTINCT] 3124 02:09:29,240 --> 02:09:30,080 >>: I LIVED IN THESE 3125 02:09:30,080 --> 02:09:32,760 QUESTIONS EACH OF THEM CAN BE 3126 02:09:32,760 --> 02:09:33,720 45 MINUTES TO ONE HOUR. 3127 02:09:33,720 --> 02:09:35,680 >>: YES, TIME IS GOING. 3128 02:09:35,680 --> 02:09:38,480 >>: I WILL TRY TO 3129 02:09:38,480 --> 02:09:39,040 SUMMARIZE. FIRST I WANT TO 3130 02:09:39,040 --> 02:09:42,160 PREFACE BY SAYING, CHRISTOPHER 3131 02:09:42,160 --> 02:09:45,360 IS A REALLY POWERFUL 3132 02:09:45,360 --> 02:09:45,920 TECHNOLOGY, THERE STILL SOME 3133 02:09:45,920 --> 02:09:48,880 QUESTIONS REGARDING HIS 3134 02:09:48,880 --> 02:09:50,320 SPECIFICITY. IF YOU TARGET OR 3135 02:09:50,320 --> 02:09:52,120 USE THIS TO REACH THE GENOME 3136 02:09:52,120 --> 02:09:54,280 YOU CAN EXCISE THAT REGION OF 3137 02:09:54,280 --> 02:09:55,680 THE GENOME AND IN THIS CASE, 3138 02:09:55,680 --> 02:09:58,120 THIS MAY BE A GOOD THING BUT 3139 02:09:58,120 --> 02:10:01,520 THAT IS A LIFELONG CHANGE. IF 3140 02:10:01,520 --> 02:10:02,680 IT'S 100% SPECIFIC AND YOU DO 3141 02:10:02,680 --> 02:10:04,480 WHAT YOU INTEND TO DO THAT IS 3142 02:10:04,480 --> 02:10:06,280 GREAT BUT THERE'S ALWAYS THE 3143 02:10:06,280 --> 02:10:07,960 QUESTION OF WHETHER OR NOT 3144 02:10:07,960 --> 02:10:09,400 IT'S DOING SOMETHING SOMEWHERE 3145 02:10:09,400 --> 02:10:11,080 ELSE AND THAT HAS BEEN 3146 02:10:11,080 --> 02:10:12,400 SOMETHING THAT, I'M NOT SURE 3147 02:10:12,400 --> 02:10:14,800 IF WE HAVE A GREAT ANSWER TO. 3148 02:10:14,800 --> 02:10:16,360 IT'S COME UP IN SOME RESPECTS 3149 02:10:16,360 --> 02:10:16,920 IN CHINA, FOR INSTANCE, WERE 3150 02:10:16,920 --> 02:10:21,480 THERE HAVE BEEN SOME STUDIES 3151 02:10:21,480 --> 02:10:24,040 DONE ON PEOPLE WHO MAY NOT 3152 02:10:24,040 --> 02:10:25,120 UNDERSTAND THE IMPLICATIONS OF 3153 02:10:25,120 --> 02:10:26,600 THESE ADVANCES. GETTING BACK 3154 02:10:26,600 --> 02:10:27,160 TO THE SPECIFIC QUESTION, 3155 02:10:27,160 --> 02:10:30,680 THERE'S BEEN A LOT OF 3156 02:10:30,680 --> 02:10:32,440 ATTENTION AND SOME VERY 3157 02:10:32,440 --> 02:10:33,880 EXCITING DATA ON USING CRISPER 3158 02:10:33,880 --> 02:10:34,480 TO EXCISE THE REPEAT. JUST TO 3159 02:10:34,480 --> 02:10:38,600 MENTION A FEW. CLAIRE AND I 3160 02:10:38,600 --> 02:10:45,480 THINK, CLEAR: AT ANOTHER 3161 02:10:45,480 --> 02:10:46,120 INSTITUTE HAS DEVELOPED A 3162 02:10:46,120 --> 02:10:48,120 GREAT MEANS OF EXCISING THE 3163 02:10:48,120 --> 02:10:52,360 REPEAT FROM HUMAN STEM CELL 3164 02:10:52,360 --> 02:10:54,520 DIRECT NEURONS AND LOOKING AT 3165 02:10:54,520 --> 02:10:54,960 THE IMPLICATIONS IT'S 3166 02:10:54,960 --> 02:10:55,560 INCREDIBLY INTERESTING BECAUSE 3167 02:10:55,560 --> 02:10:59,280 DEPENDING ON HOW YOU DO IT AND 3168 02:10:59,280 --> 02:11:01,040 WHAT EXCISE YOU GET DIFFERENT 3169 02:11:01,040 --> 02:11:01,960 RESULTS, SOME OF WHICH ARE 3170 02:11:01,960 --> 02:11:02,560 UNANTICIPATED WHICH IS WHY WE 3171 02:11:02,560 --> 02:11:06,240 NEED THIS WORK. JUST A FEW 3172 02:11:06,240 --> 02:11:09,720 DAYS AGO I THINK THERE WAS A 3173 02:11:09,720 --> 02:11:10,840 STUDY OUT FOR CHRISTIAN 3174 02:11:10,840 --> 02:11:11,280 MUELLER'S GROUP AT THE 3175 02:11:11,280 --> 02:11:11,880 UNIVERSITY OF MASSACHUSETTS 3176 02:11:11,880 --> 02:11:16,320 LOOKING AT THIS AND SEE NINE 3177 02:11:16,320 --> 02:11:16,880 REPEAT IN MODEL SYSTEMS, 3178 02:11:16,880 --> 02:11:20,880 INCLUDING TRANSGENIC MICE AND 3179 02:11:20,880 --> 02:11:22,240 HUMAN STEM CELL DIRECT 3180 02:11:22,240 --> 02:11:22,800 NEURONS. THIS INVOLVES MANY 3181 02:11:22,800 --> 02:11:26,960 PEOPLE ON THIS CALL TODAY. I 3182 02:11:26,960 --> 02:11:27,840 THINK THIS IS MOVING QUICKLY, 3183 02:11:27,840 --> 02:11:29,560 AND EVEN IF WE DO NOT USE 3184 02:11:29,560 --> 02:11:33,160 CRISPER TO EXCISE REPEATS. I 3185 02:11:33,160 --> 02:11:36,720 THINK OTHER SCIENTISTS HAVE 3186 02:11:36,720 --> 02:11:38,680 USE CRISPER TO TARGET THE 3187 02:11:38,680 --> 02:11:41,160 EXPANDED REPEATS OF RNA THAT 3188 02:11:41,160 --> 02:11:42,880 IS MADE FROM THE EXPANDED C9 3189 02:11:42,880 --> 02:11:44,840 LOCUST AND THAT HAS SHOWED 3190 02:11:44,840 --> 02:11:46,680 SOME PROMISING RESULTS TOO. 3191 02:11:46,680 --> 02:11:47,960 THIS IS AN AREA OF ACTIVE 3192 02:11:47,960 --> 02:11:48,560 RESEARCH AND HOPEFULLY WE WILL 3193 02:11:48,560 --> 02:11:52,240 SEE SOON. 3194 02:11:52,240 --> 02:11:53,560 >>: SAMMY THERE'S A FEW 3195 02:11:53,560 --> 02:11:54,520 OF THE QUESTIONS IN A BEACON 3196 02:11:54,520 --> 02:11:56,160 ANSWER BECAUSE OF YOUR 3197 02:11:56,160 --> 02:11:58,800 AUTHORITY IS RELATED TO THAT. 3198 02:11:58,800 --> 02:11:59,280 IT'S TOO ELABORATES ON 3199 02:11:59,280 --> 02:12:01,960 APPROACHES TO STUDY DARK 3200 02:12:01,960 --> 02:12:03,120 MATTER [INDISTINCT] IS A 3201 02:12:03,120 --> 02:12:07,920 GENERAL SET OF APPROACHES? I 3202 02:12:07,920 --> 02:12:08,680 KNOW THEY HAVE THAT STUDY SO 3203 02:12:08,680 --> 02:12:13,560 MAYBE QUICKLY IF YOU CAN. 3204 02:12:13,560 --> 02:12:14,920 >>: I CAN TOUCH ON IT A 3205 02:12:14,920 --> 02:12:15,440 LITTLE BIT BECAUSE I THINK 3206 02:12:15,440 --> 02:12:20,280 PRIMARILY RIGHT NOW WE ARE IN 3207 02:12:20,280 --> 02:12:22,960 A PHASE THAT'S FOCUSED ON 3208 02:12:22,960 --> 02:12:24,920 EPIDEMIOLOGY. IT'S REALLY 3209 02:12:24,920 --> 02:12:26,880 USING DATA THAT'S AVAILABLE TO 3210 02:12:26,880 --> 02:12:28,080 US TO IDENTIFY THOSE EXPOSURES 3211 02:12:28,080 --> 02:12:29,320 THAT ARE MOST CLOSELY EXPOSURE 3212 02:12:29,320 --> 02:12:32,600 THE RISK OF DEVELOPING ALS. I 3213 02:12:32,600 --> 02:12:33,960 GUESS HERE IN MICHIGAN WE HAVE 3214 02:12:33,960 --> 02:12:35,160 THE DUBIOUS DISTINCTION OF 3215 02:12:35,160 --> 02:12:35,760 HAVING ONE OF THE HIGHEST 3216 02:12:35,760 --> 02:12:39,120 RATES OF ALS IN THE COUNTRY. 3217 02:12:39,120 --> 02:12:43,960 MAYBE THAT'S BECAUSE OF THE 3218 02:12:43,960 --> 02:12:44,840 SUPERFUND STATES SO A LOT OF 3219 02:12:44,840 --> 02:12:45,880 TOXINS AND TRASH HAS BEEN 3220 02:12:45,880 --> 02:12:48,200 DUMPED HERE AND PESTICIDES. 3221 02:12:48,200 --> 02:12:50,680 GREAT PLACE TO LIVE, IT IS 3222 02:12:50,680 --> 02:12:51,480 VERY BEAUTIFUL BUT THERE'S A 3223 02:12:51,480 --> 02:12:51,960 SLIGHTLY HIGH RISK OF 3224 02:12:51,960 --> 02:12:57,160 DEVELOPING ALS. TRYING TO 3225 02:12:57,160 --> 02:12:59,400 TURN AROUND AND USE AS AN 3226 02:12:59,400 --> 02:13:00,880 ADVANTAGE TO IDENTIFY THE 3227 02:13:00,880 --> 02:13:01,800 SPECIFIC EXPOSURE THAT ARE 3228 02:13:01,800 --> 02:13:03,120 ASSOCIATED WITH ALS. THAT IN 3229 02:13:03,120 --> 02:13:08,320 TURN NEEDS TO MOVE BACK INTO 3230 02:13:08,320 --> 02:13:13,240 THE AND OUTCOMES. LIKE I 3231 02:13:13,240 --> 02:13:14,360 SAID, THAT IS ONE OF THE 3232 02:13:14,360 --> 02:13:18,440 PURPOSES OF THE TRANSFORMATIVE 3233 02:13:18,440 --> 02:13:20,640 AWARD. 3234 02:13:20,640 --> 02:13:21,160 >>: THANK YOU SAMMY. 3235 02:13:21,160 --> 02:13:25,280 >>: THANK YOU. SO THIS 3236 02:13:25,280 --> 02:13:25,840 IS A QUESTION FOR LYNN AND 3237 02:13:25,840 --> 02:13:28,680 MICHAEL. HUGH IS RESPONSIBLE 3238 02:13:28,680 --> 02:13:30,680 FOR CONFIRMATORY STUDIES AND 3239 02:13:30,680 --> 02:13:34,400 ON BIOMARKERS? MANY PAPERS 3240 02:13:34,400 --> 02:13:37,280 HAVE BEEN ALTERED IN THE PAST 3241 02:13:37,280 --> 02:13:37,720 DECADE ALL ENDING WITH 3242 02:13:37,720 --> 02:13:38,240 CONFIRMATION NEEDED AND 3243 02:13:38,240 --> 02:13:39,880 SEEMINGLY NOTHING HAPPENS AND 3244 02:13:39,880 --> 02:13:42,000 NO CONSENSUS ON BIOMARKERS HAS 3245 02:13:42,000 --> 02:13:44,200 BEEN MADE. LIKELY IT'S NOT 3246 02:13:44,200 --> 02:13:44,920 TESTING OR TRUSTING ANY 3247 02:13:44,920 --> 02:13:45,560 BIOMARKER. IN SO TO ENTER 3248 02:13:45,560 --> 02:13:50,200 THIS GOPHERS? 3249 02:13:50,200 --> 02:13:50,760 >>: SURE. I THINK IT 3250 02:13:50,760 --> 02:13:53,960 LARGELY DEPENDS ON THE 3251 02:13:53,960 --> 02:13:54,560 BIOMARKER IN AND OF ITSELF. 3252 02:13:54,560 --> 02:13:57,960 WE ALREADY HEARD ON NARROW 3253 02:13:57,960 --> 02:13:58,880 FILAMENT LIGHT WHICH IS A WELL 3254 02:13:58,880 --> 02:14:04,200 ACCEPTED MARKER FOR NEURAL 3255 02:14:04,200 --> 02:14:05,840 ACCIDENTAL INJURY. IT HAS 3256 02:14:05,840 --> 02:14:09,240 BEEN HIGHLY WORKED UP AN ALS 3257 02:14:09,240 --> 02:14:09,720 PATIENT COHORTS. BOTH 3258 02:14:09,720 --> 02:14:11,320 SPORADIC AS WELL AS 3259 02:14:11,320 --> 02:14:15,120 GENETICALLY DEFINED ALS GROUPS 3260 02:14:15,120 --> 02:14:16,080 C9 AND SOD ONE. THERE I THINK 3261 02:14:16,080 --> 02:14:20,800 THAT GIVEN THE PLATFORM, SINCE 3262 02:14:20,800 --> 02:14:21,680 THIS IS A WIDELY AND IS A 3263 02:14:21,680 --> 02:14:23,560 COMMERCIALLY AVAILABLE KIDS IT 3264 02:14:23,560 --> 02:14:28,280 IS WELL VALIDATED THAT THOSE 3265 02:14:28,280 --> 02:14:29,120 RESULTS, EVEN THOUGH THEY 3266 02:14:29,120 --> 02:14:31,120 MIGHT BE PRODUCED BY 3267 02:14:31,120 --> 02:14:32,920 INDEPENDENT GROUPS AT VARIOUS 3268 02:14:32,920 --> 02:14:33,760 INSTITUTIONS IN BOTH ACADEMIA 3269 02:14:33,760 --> 02:14:35,440 AS WELL AS AN INDUSTRY. YOU 3270 02:14:35,440 --> 02:14:37,560 HAVE HIGH CONFIDENCE THAT FOR 3271 02:14:37,560 --> 02:14:38,240 THE SET BIOMARKER IT REALLY 3272 02:14:38,240 --> 02:14:44,560 HAS EMERGED AND IS WELL 3273 02:14:44,560 --> 02:14:49,960 VALIDATED. IN ADDITION, SOME 3274 02:14:49,960 --> 02:14:51,040 CLINICAL SITES INCLUDING MAYO 3275 02:14:51,040 --> 02:14:51,560 CLINIC FOR EXAMPLE NOW ARE 3276 02:14:51,560 --> 02:14:55,320 OFFERING THAT TO PATIENTS AS 3277 02:14:55,320 --> 02:14:59,920 THEY GET THEIR ALS WORK UP. 3278 02:14:59,920 --> 02:15:01,600 IT'S NOT NEEDED OBVIOUSLY FOR 3279 02:15:01,600 --> 02:15:04,120 THE DIAGNOSIS FOR ALS BUT I 3280 02:15:04,120 --> 02:15:06,280 THINK NOW SOME CLINICIANS AND 3281 02:15:06,280 --> 02:15:07,160 NEUROMUSCULAR AS WELL AS 3282 02:15:07,160 --> 02:15:08,080 BEHAVIORAL NEUROLOGY INCLUDING 3283 02:15:08,080 --> 02:15:14,120 THAT AS A MEANS TO FOLLOW NFL 3284 02:15:14,120 --> 02:15:14,920 LEVELS DURING PROGRESSION. 3285 02:15:14,920 --> 02:15:17,320 NOT ONLY FOR CLINICAL BUT 3286 02:15:17,320 --> 02:15:20,400 PERHAPS FOR RESEARCH AND 3287 02:15:20,400 --> 02:15:20,960 POINT. I THINK WERE THINGS 3288 02:15:20,960 --> 02:15:23,360 GET A LITTLE BIT, AND THIS MAY 3289 02:15:23,360 --> 02:15:27,640 BE BECAUSE YOUR QUESTION IS 3290 02:15:27,640 --> 02:15:30,000 FOR NEWER TARGETS, SOME 3291 02:15:30,000 --> 02:15:32,480 TARGETS ARE DEVELOPED BY 3292 02:15:32,480 --> 02:15:33,040 COMPANIES FOR EXAMPLE FOR 3293 02:15:33,040 --> 02:15:34,240 TARGETING ENGAGEMENTS. IS 3294 02:15:34,240 --> 02:15:36,080 THERE A DRUG GETTING INTO THE 3295 02:15:36,080 --> 02:15:37,520 BRAIN AND HAVING A THERAPEUTIC 3296 02:15:37,520 --> 02:15:39,400 EFFECT? FOR THOSE TYPES OF 3297 02:15:39,400 --> 02:15:43,600 BIOMARKERS THEY MIGHT BE 3298 02:15:43,600 --> 02:15:45,320 DEVELOPED BY A SPECIFIC GROUP 3299 02:15:45,320 --> 02:15:49,600 AND THOSE FREE AGENTS WOULD BE 3300 02:15:49,600 --> 02:15:50,280 SPECIFIC TO THAT LABORATORY OR 3301 02:15:50,280 --> 02:15:51,000 INSTITUTIONS. THERE COMES A 3302 02:15:51,000 --> 02:15:52,000 LITTLE BIT MORE CHALLENGING. 3303 02:15:52,000 --> 02:15:57,560 BY IN LARGE, FOUR NOVEL 3304 02:15:57,560 --> 02:16:01,080 BIOMARKERS IT COMES THROUGH 3305 02:16:01,080 --> 02:16:02,800 THAT THE COOPERATIVE EFFORTS 3306 02:16:02,800 --> 02:16:04,040 IN THOSE FREE AGENTS ARE 3307 02:16:04,040 --> 02:16:06,720 SHARED AND WE HAVE HAD GREAT 3308 02:16:06,720 --> 02:16:10,120 TRACK RECORD AS A GROUP FOR 3309 02:16:10,120 --> 02:16:10,640 THIS. ONE WOULD BE THE 3310 02:16:10,640 --> 02:16:14,200 DEVELOPMENT OF A POLY GDP FOR 3311 02:16:14,200 --> 02:16:18,400 C9 AND HOW PATIENTS, AS WELL 3312 02:16:18,400 --> 02:16:21,880 AS THE BIO FLUIDS OF THAT WE 3313 02:16:21,880 --> 02:16:23,880 ARE SHARED TO DEVELOP 3314 02:16:23,880 --> 02:16:25,040 MEANINGFUL COHORTS INVALIDATED 3315 02:16:25,040 --> 02:16:26,760 COHORTS. THIS WAS ACHIEVED SO 3316 02:16:26,760 --> 02:16:28,560 I THINK FOR NEWER BIOMARKERS 3317 02:16:28,560 --> 02:16:29,160 TO SPECIFICALLY ANSWER YOUR 3318 02:16:29,160 --> 02:16:31,160 QUESTION, I THINK IT'S 3319 02:16:31,160 --> 02:16:33,800 CHALLENGING BECAUSE THEY ARE 3320 02:16:33,800 --> 02:16:35,200 NOT A COMMERCIALLY AVAILABLE 3321 02:16:35,200 --> 02:16:35,800 KIT. IT WOULD REQUIRE FURTHER 3322 02:16:35,800 --> 02:16:37,760 OPTIMIZATIONS OF THOSE AND 3323 02:16:37,760 --> 02:16:42,240 VALIDATION OF OTHER 3324 02:16:42,240 --> 02:16:43,480 INDEPENDENT COHORTS TO REALLY 3325 02:16:43,480 --> 02:16:44,280 INCREASE YOUR CONFIDENCE THAT 3326 02:16:44,280 --> 02:16:44,880 WHAT YOU'RE SEEING WITH THIS 3327 02:16:44,880 --> 02:16:49,680 MARKER IS IN FACT MEANINGFUL. 3328 02:16:49,680 --> 02:16:50,080 MICHAEL. 3329 02:16:50,080 --> 02:16:51,720 >>: TO ADD ON TO WHAT WAS 3330 02:16:51,720 --> 02:16:54,520 SAID, I THINK NARROW FILAMENT 3331 02:16:54,520 --> 02:16:55,000 HAS BEEN AN UNBELIEVABLY 3332 02:16:55,000 --> 02:16:56,760 USEFUL BIOMARKER. I THINK ANY 3333 02:16:56,760 --> 02:16:59,960 SERIOUS STUDY INCLUDES THIS AS 3334 02:16:59,960 --> 02:17:02,960 A COMMON READOUTS. ONE OF THE 3335 02:17:02,960 --> 02:17:04,560 CHALLENGES HOWEVER IS THAT A, 3336 02:17:04,560 --> 02:17:05,600 IT'S NOT SPECIFIC. AS SEEN IN 3337 02:17:05,600 --> 02:17:08,040 ALS AS WELL AS A RANGE OF ORAL 3338 02:17:08,040 --> 02:17:10,800 GENERATIVE DISORDERS BECAUSE 3339 02:17:10,800 --> 02:17:12,320 ITS RELEASE AS THEY DO THIS. 3340 02:17:12,320 --> 02:17:13,600 THAT'S A RELATIVELY LATE 3341 02:17:13,600 --> 02:17:15,240 BIOMARKER BUT IS STILL REALLY 3342 02:17:15,240 --> 02:17:17,240 USEFUL. ONE CAN SEE FROM 3343 02:17:17,240 --> 02:17:18,040 TRIALS THAT THE NEURAL 3344 02:17:18,040 --> 02:17:19,880 FILAMENT LEVELS CHANGE IN 3345 02:17:19,880 --> 02:17:21,360 THOSE RETREATED VERSUS 3346 02:17:21,360 --> 02:17:22,160 UNTREATED. IT IS VERY CLEAR 3347 02:17:22,160 --> 02:17:24,280 EVEN BEFORE CLINICAL DATA CAME 3348 02:17:24,280 --> 02:17:26,200 BACK TO THIS DRUG WAS WORKING. 3349 02:17:26,200 --> 02:17:28,080 I THINK THAT'S AN ILLUSTRATION 3350 02:17:28,080 --> 02:17:29,960 OF HOW THAT BIOMARKER HAS 3351 02:17:29,960 --> 02:17:31,640 ALREADY PROVEN ITS WORTH. BUT 3352 02:17:31,640 --> 02:17:32,880 WE NEED TO MOVE FURTHER 3353 02:17:32,880 --> 02:17:34,720 UPSTREAM AND WE NEED TO START 3354 02:17:34,720 --> 02:17:35,200 MOVING TOWARDS MOLECULAR 3355 02:17:35,200 --> 02:17:42,760 TARGETS AS IT WAS SAID. BUT I 3356 02:17:42,760 --> 02:17:44,080 ALSO THINK THERE'S A LOT OF 3357 02:17:44,080 --> 02:17:45,240 EXCITEMENT AND ONGOING EFFORTS 3358 02:17:45,240 --> 02:17:47,560 TO DEVELOP BIOMARKERS, THAT'S 3359 02:17:47,560 --> 02:17:49,800 A PROTEIN THAT IS LOCALIZED IN 3360 02:17:49,800 --> 02:17:52,920 90% OF ALS. THERE'S BEEN A 3361 02:17:52,920 --> 02:17:54,480 NUMBER OF ATTEMPTS TO MAKE 3362 02:17:54,480 --> 02:17:56,320 BIOMARKERS TO EITHER MONITOR 3363 02:17:56,320 --> 02:17:57,000 THE AGGREGATION STATUS PROTEIN 3364 02:17:57,000 --> 02:17:57,880 BUT INCREASINGLY TO LOOK AT 3365 02:17:57,880 --> 02:17:59,600 WHAT HAPPENS WHEN IT'S NOT 3366 02:17:59,600 --> 02:18:04,280 FUNCTIONAL. OUR TEAM HAS BEEN 3367 02:18:04,280 --> 02:18:05,960 DEVELOPING THIS IN EARLY DAYS 3368 02:18:05,960 --> 02:18:07,160 BUT I THINK, FINALLY BECAUSE 3369 02:18:07,160 --> 02:18:08,960 OF THE EFFORTS OF OTHER GROUPS 3370 02:18:08,960 --> 02:18:11,280 WE KNOW HOW TO DESIGN HIS 3371 02:18:11,280 --> 02:18:11,760 BIOMARKERS. I THINK THE 3372 02:18:11,760 --> 02:18:15,640 FUTURE IS BRIGHT FOR FTD ALS 3373 02:18:15,640 --> 02:18:17,600 BIOMARKERS, BUT I THINK IT'S 3374 02:18:17,600 --> 02:18:18,560 EARLY DAYS. 3375 02:18:18,560 --> 02:18:19,160 >>: THANK YOU MICHAEL AND 3376 02:18:19,160 --> 02:18:19,840 WHILE YOU'RE ANSWERING, THERE 3377 02:18:19,840 --> 02:18:21,760 IS A QUESTION IN THE CHAT I 3378 02:18:21,760 --> 02:18:25,320 WONDER IF YOU COULD ADDRESSES 3379 02:18:25,320 --> 02:18:28,280 IS, IT IS GREAT TO FIND NEW 3380 02:18:28,280 --> 02:18:29,760 PATHWAYS AND DRUG TOPICS BUT 3381 02:18:29,760 --> 02:18:31,080 HOW WILL HE BALANCE RESEARCH 3382 02:18:31,080 --> 02:18:33,120 FOR NEW PATHWAYS IF WE ARE 3383 02:18:33,120 --> 02:18:34,680 TURNING TO PATHWAYS OF PURITY 3384 02:18:34,680 --> 02:18:35,160 FOUND IN TREATMENTS? WE 3385 02:18:35,160 --> 02:18:36,720 CLEARLY HAVE TO DO BOTH AT THE 3386 02:18:36,720 --> 02:18:38,080 SAME TIME TO WHAT IS THE RIGHT 3387 02:18:38,080 --> 02:18:39,560 BALANCE? DUE TO A SHORTAGE OF 3388 02:18:39,560 --> 02:18:43,120 RIGHT NOW? 3389 02:18:43,120 --> 02:18:43,960 >>: THIS IS A GREAT 3390 02:18:43,960 --> 02:18:45,520 QUESTION AND A HARD QUESTION 3391 02:18:45,520 --> 02:18:47,480 IS NUMBER OF APPROACHES. IF 3392 02:18:47,480 --> 02:18:49,480 YOU LOOK AT SOD ONE DISCOVERED 3393 02:18:49,480 --> 02:18:52,320 IN 1990 THREE, AND IN 2022, 3394 02:18:52,320 --> 02:18:54,360 NEARLY THREE DECADES LATER, IT 3395 02:18:54,360 --> 02:18:56,480 SEEMS WE HAVE AN EFFECTIVE 3396 02:18:56,480 --> 02:18:57,400 THERAPY. I THINK THE PATH 3397 02:18:57,400 --> 02:18:58,840 TOWARDS DRUG DEVELOPMENT IS 3398 02:18:58,840 --> 02:18:59,920 SHORTENING * SHORTENING AS WE 3399 02:18:59,920 --> 02:19:01,120 GET BETTER MODELS LIKE STEM 3400 02:19:01,120 --> 02:19:02,280 CELL MODELS. BUT IT'S STILL 3401 02:19:02,280 --> 02:19:04,120 NOT GOING TO HAPPEN OVERNIGHT. 3402 02:19:04,120 --> 02:19:05,920 WE NEED TO IDENTIFY THOSE 3403 02:19:05,920 --> 02:19:07,880 TARGETS IN A MEAN TO ENGAGE 3404 02:19:07,880 --> 02:19:09,320 PHARMA. ACADEMIC IS NOT GOOD 3405 02:19:09,320 --> 02:19:11,080 AT DEVELOPING DRUGS, WE NEEDED 3406 02:19:11,080 --> 02:19:12,840 THEM ON BOARD. THEY GET 3407 02:19:12,840 --> 02:19:13,840 EXCITED WHEN THEY SEE 3408 02:19:13,840 --> 02:19:15,760 MECHANISTIC DATA THAT IS 3409 02:19:15,760 --> 02:19:16,840 COMPELLING TO WHAT THEY WANT 3410 02:19:16,840 --> 02:19:18,160 TO INVEST HUNDREDS AND 3411 02:19:18,160 --> 02:19:19,480 MILLIONS OR BILLIONS OF 3412 02:19:19,480 --> 02:19:21,400 DOLLARS INTO THERAPY. I THINK 3413 02:19:21,400 --> 02:19:23,200 THAT SOMETHING THAT WE NEED TO 3414 02:19:23,200 --> 02:19:25,200 DO TO CONVINCE AND COMPEL THEM 3415 02:19:25,200 --> 02:19:27,920 TO BE PARTNERS. THAT'S GOING 3416 02:19:27,920 --> 02:19:28,920 TO HELP WITH TARGETS THAT HAVE 3417 02:19:28,920 --> 02:19:30,400 BEEN IDENTIFIED BUT IT IS 3418 02:19:30,400 --> 02:19:33,360 ACADEMIA AND IT IS THE MOST 3419 02:19:33,360 --> 02:19:34,680 BASIC OF SCIENCES THAT 3420 02:19:34,680 --> 02:19:36,200 IDENTIFY THOSE NEW TARGETS IS 3421 02:19:36,200 --> 02:19:37,080 ABSOLUTELY CRITICAL TO DO 3422 02:19:37,080 --> 02:19:38,480 THAT. IT'S ONLY BEEN SEVERAL 3423 02:19:38,480 --> 02:19:41,560 YEARS WE KNOW THAT TV FOR 3424 02:19:41,560 --> 02:19:42,720 THREE MISS SPLICING IS A 3425 02:19:42,720 --> 02:19:43,720 CORNERSTONE OF ALS 3426 02:19:43,720 --> 02:19:44,920 PATHOGENESIS. THAT IS A 3427 02:19:44,920 --> 02:19:46,480 BRAND-NEW TARGET THAT WE NEED 3428 02:19:46,480 --> 02:19:47,560 TO CONTINUE TO PURSUE. I 3429 02:19:47,560 --> 02:19:49,520 THINK ANY OF US WOULD BE NAÏVE 3430 02:19:49,520 --> 02:19:50,840 TO CONTINUE TO THEIR SUDDENLY 3431 02:19:50,840 --> 02:19:52,640 BIG SURPRISES IN THE ALS FIELD 3432 02:19:52,640 --> 02:19:55,040 WITH THINGS AND IT BEING VERY 3433 02:19:55,040 --> 02:19:56,320 IMPORTANT, VERY UPSTREAM, AND 3434 02:19:56,320 --> 02:19:57,480 VERY TARGETABLE. I THINK WE 3435 02:19:57,480 --> 02:19:58,800 NEED TO DO BOTH WITH THE WAY 3436 02:19:58,800 --> 02:20:00,760 THAT WE DO THAT AS WE CONTINUE 3437 02:20:00,760 --> 02:20:02,480 TO GET MORE FUNDING, BRINGING 3438 02:20:02,480 --> 02:20:04,640 PEOPLE FROM THE OUTSIDE TO GET 3439 02:20:04,640 --> 02:20:06,160 MORE RESEARCHERS, WE INVOLVE 3440 02:20:06,160 --> 02:20:07,960 PHARMA TO BROADEN THE NUMBER 3441 02:20:07,960 --> 02:20:10,880 OF GROUPS ARE INVESTED AND 3442 02:20:10,880 --> 02:20:12,320 ENGAGED. TAKE YOU VERY MUCH 3443 02:20:12,320 --> 02:20:12,920 DOCTOR MICHAEL. 3444 02:20:12,920 --> 02:20:14,840 >>: YES, HAVE A QUESTION 3445 02:20:14,840 --> 02:20:16,080 WHICH IS, WHO IS RESPONSIBLE 3446 02:20:16,080 --> 02:20:18,720 FOR SOME ACQUISITION OF GENE 3447 02:20:18,720 --> 02:20:19,200 TESTING? EACH LAB TEST 3448 02:20:19,200 --> 02:20:24,400 DIFFERENT SETS OF GENES. 3449 02:20:24,400 --> 02:20:25,000 >>: IS AN EXCELLENT 3450 02:20:25,000 --> 02:20:25,800 QUESTION BECAUSE I'M GLAD IT'S 3451 02:20:25,800 --> 02:20:28,480 BEING ASKED BECAUSE THERE IS A 3452 02:20:28,480 --> 02:20:29,040 VERY SPECIFIC EFFORT 3453 02:20:29,040 --> 02:20:29,600 HAPPENING. ACTUALLY BY THE 3454 02:20:29,600 --> 02:20:32,760 NIH ECONOMY OF THIS CLINICAL 3455 02:20:32,760 --> 02:20:36,280 GENE GNOME INTERNSHIP. THAT 3456 02:20:36,280 --> 02:20:37,960 IS FOR ALS BUT FOR MANY 3457 02:20:37,960 --> 02:20:39,880 DISEASES. AS A FORMATION OF 3458 02:20:39,880 --> 02:20:41,600 EXPERT PANEL, THERE IS IN ALS 3459 02:20:41,600 --> 02:20:43,520 EXPERTS PANEL AND WE ARE LED 3460 02:20:43,520 --> 02:20:44,760 BY OUR FEARLESS LEADER, MATT 3461 02:20:44,760 --> 02:20:49,480 HARMS, ALSO FROM COLUMBIA. HE 3462 02:20:49,480 --> 02:20:52,520 HAS INVITED A DIVERSE GROUP OF 3463 02:20:52,520 --> 02:20:53,160 EXPERTS ACROSS THE GLOBE AND 3464 02:20:53,160 --> 02:20:56,560 WE MEET TWICE A MONTH AND WE 3465 02:20:56,560 --> 02:20:58,520 GO OVER EVERY SINGLE GENE THAT 3466 02:20:58,520 --> 02:20:59,320 WAS PROPOSED TO BE ASSOCIATED 3467 02:20:59,320 --> 02:21:04,640 WITH ALS. AND I SAY PROPOSED 3468 02:21:04,640 --> 02:21:06,560 BECAUSE SOME OF IT IS NOT REAL 3469 02:21:06,560 --> 02:21:07,200 ASSOCIATIONS, THE SAMPLE SIZES 3470 02:21:07,200 --> 02:21:11,160 ARE SMALL DESPITE IT BEING 3471 02:21:11,160 --> 02:21:11,920 PUBLISHED. WE GO THROUGH 3472 02:21:11,920 --> 02:21:13,880 EVERY GENE AND WE AS A 3473 02:21:13,880 --> 02:21:15,480 COMMUNITY DECIDE IS THIS GENE, 3474 02:21:15,480 --> 02:21:16,080 SHOULD WE RECOMMEND TESTING OF 3475 02:21:16,080 --> 02:21:21,320 THIS GENE? WE HOPE THAT OUR 3476 02:21:21,320 --> 02:21:23,560 EFFORTS WOULD THEN BE DIGESTED 3477 02:21:23,560 --> 02:21:24,520 BY COMMERCIAL COMPANIES AS 3478 02:21:24,520 --> 02:21:26,360 WELL AS ACADEMIC LABS TO 3479 02:21:26,360 --> 02:21:27,280 MODIFY THEIR GENE PANELS. 3480 02:21:27,280 --> 02:21:31,000 THAT WAY IT IS STANDARDIZED. 3481 02:21:31,000 --> 02:21:31,760 AND ACTUALLY GOING TO POST AND 3482 02:21:31,760 --> 02:21:34,800 I WILL FIND A WAY TO POST THE 3483 02:21:34,800 --> 02:21:36,560 LINK TO OUR EFFORTS SO FAR. 3484 02:21:36,560 --> 02:21:38,960 WE HAVE BEEN MEETING SINCE 3485 02:21:38,960 --> 02:21:41,280 2020 AND WE HAVE A LIST. FOR 3486 02:21:41,280 --> 02:21:43,000 EVERY GENUS BEEN PROPOSED TO 3487 02:21:43,000 --> 02:21:44,720 BE ASSOCIATED WITH ALS, AS 3488 02:21:44,720 --> 02:21:46,840 THEIR DEFINITIVE EVIDENCE, AND 3489 02:21:46,840 --> 02:21:49,640 WE USED A VERY STRICT 3490 02:21:49,640 --> 02:21:50,240 CRITERIA, SOMETHING LIKE SOD 3491 02:21:50,240 --> 02:21:53,040 ONE. WE'VE ALL AGREED THAT IT 3492 02:21:53,040 --> 02:21:54,680 IS A BONA FIDE ALS GENE AND 3493 02:21:54,680 --> 02:21:55,680 THIS SHOULD BE ON EVERY SINGLE 3494 02:21:55,680 --> 02:21:59,760 PANEL AS WELL AS C9 AND MANY 3495 02:21:59,760 --> 02:22:02,160 OTHERS. THERE'S OTHER ALS 3496 02:22:02,160 --> 02:22:04,280 GENES THAT FAILED THESE VERY 3497 02:22:04,280 --> 02:22:08,040 RIGOROUS AND AGREED-UPON 3498 02:22:08,040 --> 02:22:09,280 CRITERIA. THOSE GENES SHOULD 3499 02:22:09,280 --> 02:22:12,320 NOT BE TESTED IN ANY PATIENTS, 3500 02:22:12,320 --> 02:22:15,040 PRESYMPTOMATIC OR NOT. OUR 3501 02:22:15,040 --> 02:22:16,320 RECOMMENDATION IS FOR THEM TO 3502 02:22:16,320 --> 02:22:19,560 BE REMOVED FROM PANELS. 3503 02:22:19,560 --> 02:22:20,800 AGAIN, WE CANNOT FORCE 3504 02:22:20,800 --> 02:22:22,840 COMPANIES TO REMOVE GENES. 3505 02:22:22,840 --> 02:22:25,440 BUT WHAT WERE TRYING TO DO IS 3506 02:22:25,440 --> 02:22:26,920 ENSURE THAT THEIR PANELS ARE 3507 02:22:26,920 --> 02:22:28,240 COMPREHENSIVE. THIS DOES NOT 3508 02:22:28,240 --> 02:22:30,960 MEAN BIG, THIS MEANS THAT 3509 02:22:30,960 --> 02:22:31,920 YOU'RE TESTING THE CORRECT 3510 02:22:31,920 --> 02:22:32,840 TARGETS. THERE WAS ANOTHER 3511 02:22:32,840 --> 02:22:35,120 QUESTION THE CHECK TO THAT WAS 3512 02:22:35,120 --> 02:22:36,760 RELATED IF YOU DON'T MIND, 3513 02:22:36,760 --> 02:22:39,120 SOMEBODY ASKED WHY DON'T WE DO 3514 02:22:39,120 --> 02:22:39,560 GENETIC TESTING FOR 3515 02:22:39,560 --> 02:22:40,920 PRESYMPTOMATIC INDIVIDUALS? 3516 02:22:40,920 --> 02:22:47,880 WE CAN. I'M ALSO A CLINICAL 3517 02:22:47,880 --> 02:22:48,640 MOLECULAR GENETICIST AND AS 3518 02:22:48,640 --> 02:22:49,840 PART OF THE RECOMMENDATIONS 3519 02:22:49,840 --> 02:22:50,400 FROM A COLLEGE IS THAT 3520 02:22:50,400 --> 02:22:52,400 INDIVIDUALS THAT HAVE A FAMILY 3521 02:22:52,400 --> 02:22:56,760 HISTORY, THEY THEMSELVES WE 3522 02:22:56,760 --> 02:22:59,520 DON'T KNOW IF THEY CARRY THE 3523 02:22:59,520 --> 02:23:01,480 GENETIC MUTATION OR NOT. FOR 3524 02:23:01,480 --> 02:23:04,080 THEM TO BE ELIGIBLE FOR THIS 3525 02:23:04,080 --> 02:23:05,040 ADULT ONSET DISORDER, THEY 3526 02:23:05,040 --> 02:23:07,520 HAVE TO BE GREATER THAN 18 3527 02:23:07,520 --> 02:23:09,360 YEARS OF AGE, THAT'S WHAT OUR 3528 02:23:09,360 --> 02:23:10,960 COLLEGE RECOMMENDS AND THEY 3529 02:23:10,960 --> 02:23:13,960 ALSO HAVE TO GO THROUGH A 3530 02:23:13,960 --> 02:23:15,040 GENETIC, PRESYMPTOMATIC 3531 02:23:15,040 --> 02:23:16,920 PROGRAM. THEY CAN'T JUST BE 3532 02:23:16,920 --> 02:23:19,560 IN A ROOM NEUROLOGIST FOR 3533 02:23:19,560 --> 02:23:21,200 EXAMPLE. ALTHOUGH MANY OF THE 3534 02:23:21,200 --> 02:23:21,640 NEUROLOGIST HAVE VERY 3535 02:23:21,640 --> 02:23:23,240 EXTENSIVE GENETIC BACKGROUND 3536 02:23:23,240 --> 02:23:25,200 OF COURSE AND I WENT TO 3537 02:23:25,200 --> 02:23:26,640 RECOGNIZE THAT. BUT THE 3538 02:23:26,640 --> 02:23:27,480 INDIVIDUAL THAT'S ABOUT TWO 3539 02:23:27,480 --> 02:23:29,160 AND WHO IS CURRENTLY 3540 02:23:29,160 --> 02:23:31,600 PRESYMPTOMATIC AND IS ABOUT TO 3541 02:23:31,600 --> 02:23:32,920 BE PRETESTED FOR THE 3542 02:23:32,920 --> 02:23:33,480 PRESENTATION THEY RECOMMEND 3543 02:23:33,480 --> 02:23:34,600 THEY TALK TO A GENETICIST AND 3544 02:23:34,600 --> 02:23:38,280 UNDERSTAND WHAT THIS MEANS. 3545 02:23:38,280 --> 02:23:40,200 UNDERSTAND WHAT POSSESSION OF 3546 02:23:40,200 --> 02:23:41,920 THIS VARIANCE THAT COULD LEAD 3547 02:23:41,920 --> 02:23:43,880 TO THEIR DISEASE MEANS. WERE 3548 02:23:43,880 --> 02:23:45,520 NOT DEALING WITH CYSTIC 3549 02:23:45,520 --> 02:23:47,840 FIBROSIS OR SOME OF THESE 3550 02:23:47,840 --> 02:23:48,680 HIGHLY PENETRANT MONOTONIC 3551 02:23:48,680 --> 02:23:50,120 DISORDERS. WHILE DEALING WITH 3552 02:23:50,120 --> 02:23:51,400 THE DISORDER, DEPENDING ON 3553 02:23:51,400 --> 02:23:52,840 YOUR SPECIFIC MUTATION THAT 3554 02:23:52,840 --> 02:23:53,440 YOU MAY CARRY, YOUR PHENOTYPE 3555 02:23:53,440 --> 02:23:58,320 MAY DIFFER. SO, THAT IS 3556 02:23:58,320 --> 02:24:00,280 ACTUALLY IN PLACE. WE CANNOT 3557 02:24:00,280 --> 02:24:01,480 OFFER THAT TO THE GENERAL 3558 02:24:01,480 --> 02:24:03,080 PUBLIC BECAUSE THE NATURE OF 3559 02:24:03,080 --> 02:24:04,440 THE DISEASE IS THE FACT THAT 3560 02:24:04,440 --> 02:24:06,800 WE NEED MORE CLINICAL 3561 02:24:06,800 --> 02:24:07,840 INFORMATION, WE NEED FAMILY 3562 02:24:07,840 --> 02:24:09,560 HISTORY AT TIMES SO FOR 3563 02:24:09,560 --> 02:24:11,120 INDIVIDUALS WITHOUT ANY FAMILY 3564 02:24:11,120 --> 02:24:12,360 HISTORY, BUT THEY THEMSELVES 3565 02:24:12,360 --> 02:24:14,960 HAVE THE DISEASE WERE ABLE TO 3566 02:24:14,960 --> 02:24:16,080 INTERPRET THAT GENETIC 3567 02:24:16,080 --> 02:24:17,560 VARIANTS CLEARLY AND FOR 3568 02:24:17,560 --> 02:24:19,720 INDIVIDUALS THAT HAVE A FAMILY 3569 02:24:19,720 --> 02:24:20,280 HISTORY AND WE KNOW EXACTLY 3570 02:24:20,280 --> 02:24:25,920 WHAT WERE LOOKING FOR, TESTING 3571 02:24:25,920 --> 02:24:26,760 FOR THOSE PRESYMPTOMATIC 3572 02:24:26,760 --> 02:24:27,360 INDIVIDUALS AS STRAIGHTFORWARD 3573 02:24:27,360 --> 02:24:29,000 AS WELL. THAT IS THE NATURE 3574 02:24:29,000 --> 02:24:30,200 OF GENETICS AND WHAT WE KNOW 3575 02:24:30,200 --> 02:24:31,360 ABOUT PENETRANCE AND WHAT WE 3576 02:24:31,360 --> 02:24:33,280 KNOW ABOUT THESE GENES THAT 3577 02:24:33,280 --> 02:24:37,560 HAVE BEEN STATED AS WELL. 3578 02:24:37,560 --> 02:24:38,080 >>: IS A HIGHLIGHTED 3579 02:24:38,080 --> 02:24:43,480 QUESTION AND THIS IS FOR THIS 3580 02:24:43,480 --> 02:24:46,920 AND AS YOU SHOW THERE HAS BEEN 3581 02:24:46,920 --> 02:24:48,120 GREAT PROGRESS WITH GENE 3582 02:24:48,120 --> 02:24:49,080 IDENTIFICATION AND THERE'S A 3583 02:24:49,080 --> 02:24:51,360 LARGE PROPORTION OF PATIENTS 3584 02:24:51,360 --> 02:24:52,600 WITH NO NONENERGY NOTE TYPES. 3585 02:24:52,600 --> 02:24:54,200 HOW MANY MARGINS HAVE YET TO 3586 02:24:54,200 --> 02:24:56,440 BE FOUND AND WHEN DO WE REACH 3587 02:24:56,440 --> 02:25:01,280 A POINT OF DIMINISHING 3588 02:25:01,280 --> 02:25:04,680 SYMPTOMS? 3589 02:25:04,680 --> 02:25:05,840 >>: SELL YOU ANSWER IF 3590 02:25:05,840 --> 02:25:06,720 YOU LIKE. 3591 02:25:06,720 --> 02:25:07,720 >>: OKAY, I GET ASKED 3592 02:25:07,720 --> 02:25:09,560 THIS QUESTION ALL THE TIME AND 3593 02:25:09,560 --> 02:25:12,080 I WANT TO COME BACK TO THE 3594 02:25:12,080 --> 02:25:13,240 COMMENTS THAT MICHAEL MADE 3595 02:25:13,240 --> 02:25:16,920 ABOUT HIS WIFE BEING IN 3596 02:25:16,920 --> 02:25:17,440 ONCOLOGISTS. ALSO AS A 3597 02:25:17,440 --> 02:25:20,720 CLINICAL MOLECULAR GENESIS, 3598 02:25:20,720 --> 02:25:22,280 PRENATAL AND CANCER ARE 3599 02:25:22,280 --> 02:25:23,280 BREAD-AND-BUTTER WE HAVE TO 3600 02:25:23,280 --> 02:25:24,960 KNOW THEM REGARDLESS OF OUR 3601 02:25:24,960 --> 02:25:25,800 SPECIALTY. IN CANCER IF YOU 3602 02:25:25,800 --> 02:25:26,400 HAVE A SPECIFIC MUTATION RIGHT 3603 02:25:26,400 --> 02:25:30,720 AWAY YOU'RE GIVING A MEDICINE 3604 02:25:30,720 --> 02:25:32,280 IF IT FITS YOUR PROFILE. 3605 02:25:32,280 --> 02:25:33,320 ROUTE RIGHT AWAY YOU'RE GIVING 3606 02:25:33,320 --> 02:25:35,320 ELIGIBILITY TOWARDS A CLINICAL 3607 02:25:35,320 --> 02:25:37,120 TRIAL IF YOU HAVE A SPECIFIC 3608 02:25:37,120 --> 02:25:38,240 GENETIC PROFILE. THAT'S JUST 3609 02:25:38,240 --> 02:25:39,480 BECAUSE WE HAVEN'T LOOKED AT 3610 02:25:39,480 --> 02:25:40,920 THESE AND SIMPLE REPEATS IN 3611 02:25:40,920 --> 02:25:42,400 CANCER, WE'VE LOOKED AT LOTS 3612 02:25:42,400 --> 02:25:44,280 OF OTHER THINGS, COPY NUMBER 3613 02:25:44,280 --> 02:25:44,880 VARIATIONS AND LOTS OF OTHER 3614 02:25:44,880 --> 02:25:48,720 THINGS THAT CAN DISRUPT THE 3615 02:25:48,720 --> 02:25:49,760 GENOME IN INDIVIDUALS WITH 3616 02:25:49,760 --> 02:25:52,000 CANCER. WE HAVE NOT LOOKED AT 3617 02:25:52,000 --> 02:25:53,880 EVERY ROCK OR UNDER EVERY ROCK 3618 02:25:53,880 --> 02:25:55,920 FOR ALS. IT MAY BE CERTAIN 3619 02:25:55,920 --> 02:25:57,560 THINGS ARE ABSOLUTELY NOT 3620 02:25:57,560 --> 02:25:59,200 ASSOCIATED WITH ALS THAT WERE 3621 02:25:59,200 --> 02:26:01,920 NOT THERE YET. SO, THERE IS A 3622 02:26:01,920 --> 02:26:03,560 POINT OF DIMINISHING RETURNS. 3623 02:26:03,560 --> 02:26:05,400 ABSOLUTELY, SOME NEW MUTATIONS 3624 02:26:05,400 --> 02:26:06,120 ARE SOME GENES THAT WE FIND 3625 02:26:06,120 --> 02:26:10,520 ARE QUITE RARE. BUT, IT WOULD 3626 02:26:10,520 --> 02:26:13,080 BE WORTHWHILE AGAIN TO COME 3627 02:26:13,080 --> 02:26:13,600 BACK TO THE IDEA USING THE 3628 02:26:13,600 --> 02:26:17,400 TECHNOLOGIES THAT'S FAIR AND 3629 02:26:17,400 --> 02:26:18,960 USING THE DATA THAT'S THERE 3630 02:26:18,960 --> 02:26:20,800 ALREADY AND APPLYING A NEW 3631 02:26:20,800 --> 02:26:23,200 PROPOSED IS TO DATA AND CF 3632 02:26:23,200 --> 02:26:24,920 COPY NUMBER VARIATIONS ARE 3633 02:26:24,920 --> 02:26:25,920 ENRICHED IN ALS. AT THE 3634 02:26:25,920 --> 02:26:27,560 DIFFERENT TYPES OF MUTATIONS 3635 02:26:27,560 --> 02:26:29,440 THAT WE SEE IN CANCER ARE THEY 3636 02:26:29,440 --> 02:26:31,120 ENDED AND RICH IN ALS SO WE 3637 02:26:31,120 --> 02:26:34,600 COULD MOVE ON AND WE CAN KIND 3638 02:26:34,600 --> 02:26:35,160 OF KNOW EXACTLY WHAT WERE 3639 02:26:35,160 --> 02:26:38,760 LOOKING FOR SO THAT WAY 3640 02:26:38,760 --> 02:26:42,080 CONTINUE TO INVEST IN 3641 02:26:42,080 --> 02:26:43,760 DIMINISHING RETURNS. I WOULD 3642 02:26:43,760 --> 02:26:47,600 LOVE TO HEAR HEAR YOUR AS 3643 02:26:47,600 --> 02:26:47,880 WELL. 3644 02:26:47,880 --> 02:26:48,440 >>: I LIKE WHAT YOU SAID 3645 02:26:48,440 --> 02:26:50,560 A LOT AND I WOULD ALSO LIKE TO 3646 02:26:50,560 --> 02:26:52,120 SEE THIS CONVERSE VIEW WHICH 3647 02:26:52,120 --> 02:26:54,880 IS THAT FOR THE RARE VARIANCE, 3648 02:26:54,880 --> 02:26:56,840 USUALLY VARIANCE OF A LARGE 3649 02:26:56,840 --> 02:26:57,920 EFFECT. THIS ALSO 3650 02:26:57,920 --> 02:26:58,840 CONTRIBUTION FROM THE VERY 3651 02:26:58,840 --> 02:26:59,360 SMALL EFFECT VARIANCE AND 3652 02:26:59,360 --> 02:27:02,960 PROBABLY EVERYBODY HAS LOTS 3653 02:27:02,960 --> 02:27:03,760 AND LOTS OF SMALL EFFECT 3654 02:27:03,760 --> 02:27:04,960 VARIANCE THAT TO MOVE THE 3655 02:27:04,960 --> 02:27:05,560 PERSON TOWARDS DEVELOPING ALS 3656 02:27:05,560 --> 02:27:07,120 BUT THAT OTHER FACTORS GO OVER 3657 02:27:07,120 --> 02:27:11,560 THE THRESHOLD. SO HOW MANY OF 3658 02:27:11,560 --> 02:27:12,640 THOSE GENES ARE YET TO BE 3659 02:27:12,640 --> 02:27:14,640 FOUND? WE DON'T KNOW. WE DID 3660 02:27:14,640 --> 02:27:16,360 A LARGE ENOUGH ASSOCIATIONS 3661 02:27:16,360 --> 02:27:17,280 STUDY HOW MANY WOULD YOU FIND? 3662 02:27:17,280 --> 02:27:21,000 PROBABLY HUNDREDS MORE. WE 3663 02:27:21,000 --> 02:27:22,880 SEE THAT AND OTHER DISEASES ET 3664 02:27:22,880 --> 02:27:25,000 CETERA, THE LARGER THE STUDY 3665 02:27:25,000 --> 02:27:27,240 THE MORE THE SMALL EFFECTS 3666 02:27:27,240 --> 02:27:29,720 THAT YOU FIND. THE QUESTION 3667 02:27:29,720 --> 02:27:31,920 BECOMES HOW IMPORTANT IS THAT 3668 02:27:31,920 --> 02:27:33,080 THERAPEUTICALLY? I THINK THEY 3669 02:27:33,080 --> 02:27:34,520 MIGHT BE SURPRISINGLY 3670 02:27:34,520 --> 02:27:35,680 IMPORTANT AND THERAPEUTICALLY. 3671 02:27:35,680 --> 02:27:39,120 IF YOU LOOK AT SOMETHING LIKE 3672 02:27:39,120 --> 02:27:39,760 HYPERCHOLESTEROLEMIA, SOME OF 3673 02:27:39,760 --> 02:27:41,160 THE EXAMPLE THAT THEY HAVE AN 3674 02:27:41,160 --> 02:27:43,000 EFFECT ON A VARIANCE THAT 3675 02:27:43,000 --> 02:27:45,520 REALLY ARE A SMALL EFFECTS BUT 3676 02:27:45,520 --> 02:27:47,360 YET THEY CAN LOWER YOUR 3677 02:27:47,360 --> 02:27:48,040 CHOLESTEROL MORE EFFICIENTLY 3678 02:27:48,040 --> 02:27:50,040 TO BE THERAPEUTIC. IT MAY BE 3679 02:27:50,040 --> 02:27:52,280 THAT EVEN THOSE COMMON 3680 02:27:52,280 --> 02:27:54,160 VARIANCE IF THERE ANY CRUCIAL 3681 02:27:54,160 --> 02:27:55,200 PART OF THE PATHWAY THEY MIGHT 3682 02:27:55,200 --> 02:27:59,080 BE WORTH INTERVENING 3683 02:27:59,080 --> 02:28:01,360 THERAPEUTICALLY. SO, I HOPE I 3684 02:28:01,360 --> 02:28:02,360 CAN TAKE THIS OPPORTUNITY TO 3685 02:28:02,360 --> 02:28:02,960 ASK THIS QUESTION ON THE LIST, 3686 02:28:02,960 --> 02:28:06,760 IS THAT OKAY? ELLEN PHILLIPS 3687 02:28:06,760 --> 02:28:08,560 HAS SAID THAT SHE WOULD LIKE 3688 02:28:08,560 --> 02:28:09,400 TO ANSWER THIS QUESTION LIVE. 3689 02:28:09,400 --> 02:28:13,200 THIS QUESTION IS FROM HADLEY. 3690 02:28:13,200 --> 02:28:14,400 DEAR FRIENDS, IN MY OPINION, 3691 02:28:14,400 --> 02:28:16,360 ONE OF THE LIMITATIONS THAT WE 3692 02:28:16,360 --> 02:28:17,560 FACE TODAY IS DUE TO OUR 3693 02:28:17,560 --> 02:28:18,600 INABILITY TO INCLUDE THE 3694 02:28:18,600 --> 02:28:20,040 BREAKING POINT OF ALS TO THE 3695 02:28:20,040 --> 02:28:22,720 BIG PICTURE. WE USUALLY LEAVE 3696 02:28:22,720 --> 02:28:23,640 THE UPPER NEURONS OF THIS 3697 02:28:23,640 --> 02:28:24,560 DISCUSSION WHAT DO YOU THINK 3698 02:28:24,560 --> 02:28:28,520 WE SHOULD DO TO CHANGE THIS? 3699 02:28:28,520 --> 02:28:29,080 >>: NOT REALLY I DON'T 3700 02:28:29,080 --> 02:28:32,400 KNOW HOW THAT HAPPENED. 3701 02:28:32,400 --> 02:28:39,000 >>: I WAS WONDERING TOO. 3702 02:28:39,000 --> 02:28:39,560 ANYBODY HERE ON THE PANEL 3703 02:28:39,560 --> 02:28:45,680 WOULD LIKE TO REFLECT ON THE 3704 02:28:45,680 --> 02:28:49,120 ROLE OF MULTI- NEURONS AND THE 3705 02:28:49,120 --> 02:28:51,000 FACT THAT WE DON'T STUDY THEM 3706 02:28:51,000 --> 02:28:58,200 AS MUCH AS WE HAVE DONE. 3707 02:28:58,200 --> 02:28:58,720 >>: OTHERWISE. THINK 3708 02:28:58,720 --> 02:29:02,480 THINK ABOUT AND GIVE A WRITTEN 3709 02:29:02,480 --> 02:29:04,080 RESPONSE. 3710 02:29:04,080 --> 02:29:05,120 >>: I COULD SAY THIS IS A 3711 02:29:05,120 --> 02:29:07,960 QUESTION BUT THIS HAS BEEN 3712 02:29:07,960 --> 02:29:11,720 IMPORTANT TO LOOK AT THIS 3713 02:29:11,720 --> 02:29:13,800 ACTUALLY QUITE CHALLENGING AND 3714 02:29:13,800 --> 02:29:17,600 I THINK IT'S INDEED A PLACE 3715 02:29:17,600 --> 02:29:19,120 AND A LOT OF THE MODELS THAT 3716 02:29:19,120 --> 02:29:20,560 WE HAVE NOT HAD NOT BEEN 3717 02:29:20,560 --> 02:29:22,880 EXPLORED AND I THINK IT'S A 3718 02:29:22,880 --> 02:29:29,440 POINT HERE THAT IS BEEN MADE. 3719 02:29:29,440 --> 02:29:30,040 >>: MICHAEL ARE YOU GOING 3720 02:29:30,040 --> 02:29:30,360 TO ANSWER? 3721 02:29:30,360 --> 02:29:32,240 >>: I WAS GOING TO THINK 3722 02:29:32,240 --> 02:29:32,840 OF EFFORTS TO BETTER 3723 02:29:32,840 --> 02:29:33,440 UNDERSTAND WHAT THE CORTEX IS 3724 02:29:33,440 --> 02:29:34,160 INVOLVED IN THE FRONTAL 3725 02:29:34,160 --> 02:29:37,680 TEMPORAL WALL WHICH WILL BE 3726 02:29:37,680 --> 02:29:38,480 ILLUMINATING. THERE'S MANY 3727 02:29:38,480 --> 02:29:39,880 EFFORTS IN THE STEM CELL FIELD 3728 02:29:39,880 --> 02:29:42,720 TO BE ABLE TO MODEL THESE 3729 02:29:42,720 --> 02:29:44,120 DIFFERENT NEURAL SUBTYPES, 3730 02:29:44,120 --> 02:29:45,320 WHICH INCLUDES UPPER MOTOR 3731 02:29:45,320 --> 02:29:46,680 NEURONS. WE DO NOT HAVE GOOD 3732 02:29:46,680 --> 02:29:47,280 CELLULAR MODELS AS OPPOSED TO 3733 02:29:47,280 --> 02:29:50,160 LOWER ONES. THAT IS AN AREA 3734 02:29:50,160 --> 02:29:57,320 OF A GAP IN OUR KNOWLEDGE. 3735 02:29:57,320 --> 02:29:57,800 >>: IF I CAN EDIT 3736 02:29:57,800 --> 02:29:58,720 SOMETHING IT'S IMPORTANT THAT 3737 02:29:58,720 --> 02:30:00,080 IT'S BEEN SAID AND IT WAS SAID 3738 02:30:00,080 --> 02:30:01,680 BEAUTIFULLY. IT WAS REALLY 3739 02:30:01,680 --> 02:30:04,280 PIONEERING THESE CHALLENGES 3740 02:30:04,280 --> 02:30:08,560 AND THEY ARE STUDYING MOTOR 3741 02:30:08,560 --> 02:30:11,000 NEURONS BUT I THINK THEY CAN 3742 02:30:11,000 --> 02:30:12,000 ABOUT NEW TECHNOLOGIES WE HAVE 3743 02:30:12,000 --> 02:30:13,840 TO TAKE ADVANTAGE REALLY OF 3744 02:30:13,840 --> 02:30:15,480 IMAGING AND IN PATIENTS TO 3745 02:30:15,480 --> 02:30:22,240 REALLY LOOK AT THE UPPER MOTOR 3746 02:30:22,240 --> 02:30:23,560 NEURON AND IN THIS PANEL AND 3747 02:30:23,560 --> 02:30:26,240 THEN OFTEN IN RESEARCH WE 3748 02:30:26,240 --> 02:30:28,400 DON'T REALLY THINK ABOUT 3749 02:30:28,400 --> 02:30:28,840 STUDIES AND THESE NEW 3750 02:30:28,840 --> 02:30:29,400 TECHNOLOGIES THAT GO BEYOND 3751 02:30:29,400 --> 02:30:33,600 THE BASIC SCIENCE. THAT IS 3752 02:30:33,600 --> 02:30:34,200 PERHAPS AN AREA WHERE WE COULD 3753 02:30:34,200 --> 02:30:38,720 REALLY INVEST MORE AND LOOK AT 3754 02:30:38,720 --> 02:30:45,160 THE PROCESSES OF UNDERGOING 3755 02:30:45,160 --> 02:30:45,480 [INDISTINCT] 3756 02:30:45,480 --> 02:30:50,920 >>: I THINK A LOT OF 3757 02:30:50,920 --> 02:30:52,120 ASPECTS OF POORLY MENTIONED 3758 02:30:52,120 --> 02:30:52,720 CLINICALLY THINGS AS WELL. WE 3759 02:30:52,720 --> 02:30:58,560 DON'T HAVE THE SAME DETAIL 3760 02:30:58,560 --> 02:31:02,720 CLINICALLY FOR THIS. SO I 3761 02:31:02,720 --> 02:31:04,400 THINK WERE VERY CLOSE TO THE 3762 02:31:04,400 --> 02:31:05,600 FINISH TIME. YOU PROBABLY 3763 02:31:05,600 --> 02:31:06,440 WILL HAVE TIME FOR THE LAST 3764 02:31:06,440 --> 02:31:09,280 QUESTION. I'M GOING TO DIRECT 3765 02:31:09,280 --> 02:31:10,440 IT TO A CLOSE THEY ARE. ALL 3766 02:31:10,440 --> 02:31:12,600 OF THE OTHER QUESTIONS WILL BE 3767 02:31:12,600 --> 02:31:15,080 ANSWERED AND WILL TAKE THEM 3768 02:31:15,080 --> 02:31:19,240 INTO ACCOUNT THIS IS ALL WE 3769 02:31:19,240 --> 02:31:22,320 HAVE TIME FOR THE SESSION. 3770 02:31:22,320 --> 02:31:23,040 THANK YOU VERY MUCH OUR 3771 02:31:23,040 --> 02:31:24,520 WORKING GROUP. IT'S BEEN 3772 02:31:24,520 --> 02:31:25,720 WONDERFUL WORKING WITH YOU ALL 3773 02:31:25,720 --> 02:31:27,760 AND IT'S BEEN PRACTICALLY 3774 02:31:27,760 --> 02:31:29,000 PLEASURABLE WORKING WITH SUCH 3775 02:31:29,000 --> 02:31:30,120 LOVELY PEOPLE. THANK YOU TO 3776 02:31:30,120 --> 02:31:30,680 THE ATTENDEES FOR SUCH GREAT 3777 02:31:30,680 --> 02:31:34,600 QUESTIONS TO GET TO MY COCHAIR 3778 02:31:34,600 --> 02:31:36,000 FOR BEING AMAZING TO WORK WITH 3779 02:31:36,000 --> 02:31:38,200 AS WELL. WE WILL CONTINUE TO 3780 02:31:38,200 --> 02:31:40,960 COLLECT QUESTIONS THROUGH THE 3781 02:31:40,960 --> 02:31:43,480 ENGAGEMENT FORM ON THE MEETING 3782 02:31:43,480 --> 02:31:44,040 WEBSITE AND LOOK FORWARD TO 3783 02:31:44,040 --> 02:31:45,760 SHAPING THE ALS STRATEGIC PLAN 3784 02:31:45,760 --> 02:31:48,040 AS YOUR PLAN IT IS SHARE 3785 02:31:48,040 --> 02:31:50,120 STRATEGIC LIGHT AND ONE OF THE 3786 02:31:50,120 --> 02:31:51,200 IMPORTANT OPPORTUNITIES TO 3787 02:31:51,200 --> 02:31:52,280 ADVANCE RESEARCH. THANK YOU 3788 02:31:52,280 --> 02:31:52,720 ALL AGAIN. 3789 02:31:52,720 --> 02:31:56,880 >>: THANK YOU SO MUCH 3790 02:31:56,880 --> 02:32:07,040 EVERYONE. 3791 02:32:08,640 --> 02:32:12,200 >>: BREAKDOWN. 3792 02:32:12,200 --> 02:32:12,760 >>: THANK YOU TO THIS 3793 02:32:12,760 --> 02:32:14,640 WORKING GROUP. THIS HAS BEEN 3794 02:32:14,640 --> 02:32:17,800 AN INCREDIBLY SIMILAR TO A 3795 02:32:17,800 --> 02:32:19,000 CONVERSATION WE HAD. THANK 3796 02:32:19,000 --> 02:32:21,200 YOU FOR ALL OF THE 3797 02:32:21,200 --> 02:32:21,720 PARTICIPANTS TO ASK THESE 3798 02:32:21,720 --> 02:32:22,240 WONDERFUL QUESTIONS AND 3799 02:32:22,240 --> 02:32:23,480 CHALLENGING QUESTIONS AS YOU 3800 02:32:23,480 --> 02:32:25,120 CAN SEE WE DON'T HAVE ANSWERS 3801 02:32:25,120 --> 02:32:28,520 FOR ALL OF THESE QUESTIONS. 3802 02:32:28,520 --> 02:32:30,440 WE NEED TO HEAR THESE 3803 02:32:30,440 --> 02:32:32,080 CHALLENGING QUESTIONS THAT WE 3804 02:32:32,080 --> 02:32:32,800 THINK REALLY ARE FORWARD AND 3805 02:32:32,800 --> 02:32:35,560 WHERE DO WE NEED TO FOCUS ON 3806 02:32:35,560 --> 02:33:01,200 NEXT 3807 02:33:01,200 --> 02:33:05,400 >>THANK YOU I'M PLEASED TO BE 3808 02:33:05,400 --> 02:33:14,840 PART OF THIS GROUP WITH STACY. 3809 02:33:14,840 --> 02:33:16,560 I'D LIKE TO BEGIN BY INTRODUCING 3810 02:33:16,560 --> 02:33:19,720 MYSELF AND THE MEMBERS AND I'LL 3811 02:33:19,720 --> 02:33:21,800 ASK THEM TO INDUCE THEMSELVES IN 3812 02:33:21,800 --> 02:33:22,160 TURN. 3813 02:33:22,160 --> 02:33:25,640 I'M NEIL SCHNEIDER. 3814 02:33:25,640 --> 02:33:30,280 THE DIRECTOR OF THE LOU GHERIG 3815 02:33:30,280 --> 02:33:32,720 CENTER AT COLUMBIA AND HEAD OF 3816 02:33:32,720 --> 02:33:40,000 MOTOR DISORDERS HERE AND I AM A 3817 02:33:40,000 --> 02:33:42,440 NEUROLOGIST I FOCUS ON THE 3818 02:33:42,440 --> 02:33:44,720 PRACTICE OF ALS SCIENTISTS AND 3819 02:33:44,720 --> 02:33:47,200 MY LABORATORY FOCUSES ON 3820 02:33:47,200 --> 02:33:50,600 UNDERSTANDING MECHANISMS OF 3821 02:33:50,600 --> 02:33:55,080 NEURODEGENERATION AND ALS AND 3822 02:33:55,080 --> 02:33:56,120 FRONTAL TEMPORAL DEMENTIA AND 3823 02:33:56,120 --> 02:33:57,600 INVOLVED IN THE DEVELOPMENT OF 3824 02:33:57,600 --> 02:33:58,760 NOVEL THERAPEUTICS LARGELY FOR 3825 02:33:58,760 --> 02:34:00,600 THE RARE GENETIC FORMS OF THE 3826 02:34:00,600 --> 02:34:05,360 DISEASE. 3827 02:34:05,360 --> 02:34:08,720 BEFORE I TURN TO STACIE, I WANT 3828 02:34:08,720 --> 02:34:13,480 TO SAY UNFORTUNATELY FRANK 3829 02:34:13,480 --> 02:34:20,720 BENNETTE AND THOMAS IS UNABLE TO 3830 02:34:20,720 --> 02:34:26,880 JOIN US. 3831 02:34:26,880 --> 02:34:28,680 THOMAS IS A PROMISING YOUNG 3832 02:34:28,680 --> 02:34:31,400 SCIENTIST AT THE UNIVERSITY OF 3833 02:34:31,400 --> 02:34:33,200 ILLINOIS CHAMPAGNE. 3834 02:34:33,200 --> 02:34:36,080 THEY'VE CONTRIBUTED EXTENSIVELY 3835 02:34:36,080 --> 02:34:39,960 TO OUR STIMULATING DISCUSS AND 3836 02:34:39,960 --> 02:34:41,080 UNFORTUNATELY UNABLE TO JOIN US 3837 02:34:41,080 --> 02:34:43,920 THIS AFTERNOON. 3838 02:34:43,920 --> 02:34:44,720 STACIE. 3839 02:34:44,720 --> 02:34:50,680 >>STACE WIE WENINGER AND Ph.D. 3840 02:34:50,680 --> 02:34:52,840 NEUROSCIENTIST AND RUN A PROGRAM 3841 02:34:52,840 --> 02:34:57,440 CALLED FBRI WHERE WE FOCUS ON 3842 02:34:57,440 --> 02:35:00,680 INVESTING AT NEURODEGENERATIVE 3843 02:35:00,680 --> 02:35:04,720 DISEASES IN ACADEMIA AND IN 3844 02:35:04,720 --> 02:35:08,000 THERAPEUTICS AND COMPANIES AND 3845 02:35:08,000 --> 02:35:11,560 RUN A WEBSITE PRIMARILY FOCUSSED 3846 02:35:11,560 --> 02:35:13,640 ON ALZHEIMER'S DISEASE AND 3847 02:35:13,640 --> 02:35:15,480 COVERS MORE NEURODEGENERATIVE 3848 02:35:15,480 --> 02:35:15,760 DISEASES. 3849 02:35:15,760 --> 02:35:21,960 IT'S A WEBSITE FOR RESEARCHERS 3850 02:35:21,960 --> 02:35:32,480 BUT FOR ANYBODY INTERESTED IN 3851 02:35:33,960 --> 02:35:34,920 NEURODEGENERATIVE DISEASES AND 3852 02:35:34,920 --> 02:35:36,800 LEARN THE LATEST THERE. 3853 02:35:36,800 --> 02:35:38,120 WE'LL TURN IT TO INTRODUCE THE 3854 02:35:38,120 --> 02:35:39,560 REST OF OUR MEMBERS. 3855 02:35:39,560 --> 02:35:43,120 JOE, DO YOU WANT TO INTRODUCE 3856 02:35:43,120 --> 02:35:43,680 YOURSELF? 3857 02:35:43,680 --> 02:35:46,360 >>HI, I'M CHIEF SCIENTIFIC 3858 02:35:46,360 --> 02:35:51,320 OFFICER AT A COMPANY WHERE 3859 02:35:51,320 --> 02:35:55,080 UNIFORMLY FOCUSSED ON COMBATTING 3860 02:35:55,080 --> 02:35:56,960 NEURODEGENERATIVE DISEASE. 3861 02:35:56,960 --> 02:36:00,160 I'VE BEEN DOING DRUG DISCOVERY 3862 02:36:00,160 --> 02:36:01,840 AND ALS SPECIFICALLY CLOSE TO 20 3863 02:36:01,840 --> 02:36:04,640 YEARS AND HAVE BEEN LUCKY TO 3864 02:36:04,640 --> 02:36:06,400 BRING THREE DIFFERENT DRUGS TO 3865 02:36:06,400 --> 02:36:07,480 CLINICAL TRIALS FOR ALS. 3866 02:36:07,480 --> 02:36:11,160 GREAT TO BE PART OF THE 3867 02:36:11,160 --> 02:36:12,720 INITIATIVE AND THANKS FOR 3868 02:36:12,720 --> 02:36:16,720 INCLUDING ME. 3869 02:36:16,720 --> 02:36:16,960 STEFFEN. 3870 02:36:16,960 --> 02:36:18,960 >>I'M THE VICE PRESIDENT AND 3871 02:36:18,960 --> 02:36:23,760 HEAD OF NEUROSCIENCE AT A 3872 02:36:23,760 --> 02:36:24,320 PRIVATELY HELD THERAPEUTIC 3873 02:36:24,320 --> 02:36:27,840 COMPANY IN BOSTON FOCUSSING ON 3874 02:36:27,840 --> 02:36:29,200 CNS DISORDER. 3875 02:36:29,200 --> 02:36:32,720 I'VE BEEN AT DRUG DISCOVERY AT 3876 02:36:32,720 --> 02:36:35,960 LARGE PHARMA AND ADJUNCT 3877 02:36:35,960 --> 02:36:36,680 PROFESSOR OF BRAIN SCIENCE AT 3878 02:36:36,680 --> 02:36:43,800 BROWN UNIVERSITY. 3879 02:36:43,800 --> 02:36:49,800 >>AND NADIA I'M HEARING FROM 3880 02:36:49,800 --> 02:36:54,920 SOME PEOPLE THEY CAN'T SEE OTHER 3881 02:36:54,920 --> 02:36:55,160 PANELISTS. 3882 02:36:55,160 --> 02:37:01,120 >>I'M NADIA SETHI I LANDED HERE 3883 02:37:01,120 --> 02:37:03,560 BECAUSE I LOST MY HUSBAND TO AN 3884 02:37:03,560 --> 02:37:06,880 AGGRESSIVE FORM OF ALS IN 2020. 3885 02:37:06,880 --> 02:37:10,000 I'M A DENTIST BY TRAINING AND 3886 02:37:10,000 --> 02:37:11,680 VERY INTERESTED IN ALS RESEARCH 3887 02:37:11,680 --> 02:37:13,720 AND BACK IN SCHOOL LEARNING MORE 3888 02:37:13,720 --> 02:37:17,320 ABOUT THE GENETICS OF ALS AND 3889 02:37:17,320 --> 02:37:18,560 I'VE BEEN INVOLVED IN A LOT OF 3890 02:37:18,560 --> 02:37:22,360 ADVOCACY INITIATIVES OVER THE 3891 02:37:22,360 --> 02:37:23,000 LAST THREE YEARS AROUND STRONG 3892 02:37:23,000 --> 02:37:32,000 SCIENCE FOR ALS. 3893 02:37:32,000 --> 02:37:34,000 >>HAPPY TO BE HERE AND WORKING 3894 02:37:34,000 --> 02:37:35,080 WITH THE COMMITTEE ON WHAT WE'LL 3895 02:37:35,080 --> 02:37:37,480 PRESENT TODAY. 3896 02:37:37,480 --> 02:37:39,640 I'M BEEN AT PENN OVER 30 YEARS 3897 02:37:39,640 --> 02:37:42,440 AND RUN THE GENE THERAPY PROGRAM 3898 02:37:42,440 --> 02:37:52,920 IN THE ORGAN DISEASE CENTER. 3899 02:38:06,240 --> 02:38:08,520 >>AND HENRIK. 3900 02:38:08,520 --> 02:38:11,320 >>I'M HENRIK ZETTERBERG AT THE 3901 02:38:11,320 --> 02:38:14,920 UNIVERSITY OF LONDON AND I 3902 02:38:14,920 --> 02:38:16,680 ENJOYED AND GOTH ENBURG AND MY 3903 02:38:16,680 --> 02:38:22,040 RESEARCH IS FOCUSSED ON FLUID 3904 02:38:22,040 --> 02:38:22,560 BIOMARKERS FROM 3905 02:38:22,560 --> 02:38:24,720 NEURODEGENERATIVE DISEASES AND 3906 02:38:24,720 --> 02:38:28,040 HAVE BEEN WORKING ON ALZHEIMER'S 3907 02:38:28,040 --> 02:38:31,040 AND FRONTAL LOBE DEMENTIAS AND 3908 02:38:31,040 --> 02:38:31,680 LOOKING FORWARD TO THE SESSION. 3909 02:38:31,680 --> 02:38:34,880 THANKS. 3910 02:38:34,880 --> 02:38:44,040 >>I'LL TAKE THE NEXT SLIDE. 3911 02:38:44,040 --> 02:38:46,240 >>STACIE WILL BE DESCRIBING IN 3912 02:38:46,240 --> 02:38:48,240 MORE DETAIL THE PRIORITIES WE 3913 02:38:48,240 --> 02:38:51,600 LAID OUT IN THE DRAFT DOCUMENT. 3914 02:38:51,600 --> 02:38:57,960 I THOUGHT I'D BEGIN BY OFFERING 3915 02:38:57,960 --> 02:39:05,160 INTRODUCTORY COMMENTS AND OUR 3916 02:39:05,160 --> 02:39:08,720 PAN DATE WAS TO TALK ABOUT AND 3917 02:39:08,720 --> 02:39:10,040 MEASURE MANDATE WAS TO MAKE 3918 02:39:10,040 --> 02:39:11,000 SUGGESTIONS AND PRIORITIES TO 3919 02:39:11,000 --> 02:39:13,520 THE NIH ABOUT HOW TO TRANSLATE 3920 02:39:13,520 --> 02:39:17,000 THE ACCELERATION OF FUNDAMENTAL 3921 02:39:17,000 --> 02:39:18,320 RESEARCH INTO EFFECTIVE ALS 3922 02:39:18,320 --> 02:39:20,720 THERAPEUTICS. 3923 02:39:20,720 --> 02:39:24,000 WHAT IS TRANSLATIONAL RESEARCH. 3924 02:39:24,000 --> 02:39:26,480 AS DEPICTED HERE IT SITS IN 3925 02:39:26,480 --> 02:39:29,480 BETWEEN AT THE INTERFACE BETWEEN 3926 02:39:29,480 --> 02:39:31,440 BASIC AND CLINICAL RESEARCH. 3927 02:39:31,440 --> 02:39:32,200 THERE'S A LOT OF OVERLAP AND 3928 02:39:32,200 --> 02:39:33,160 THAT'S REFLECTED IN THE DRAFT 3929 02:39:33,160 --> 02:39:35,920 DOCUMENT. 3930 02:39:35,920 --> 02:39:37,360 AS WE HEARD FROM OUR BASIC 3931 02:39:37,360 --> 02:39:39,040 SCIENCE COLLEAGUES THIS MORNING, 3932 02:39:39,040 --> 02:39:40,720 BASIC RESEARCH REALLY IS 3933 02:39:40,720 --> 02:39:42,520 FOCUSSED ON UNDERSTANDING 3934 02:39:42,520 --> 02:39:44,800 MECHANISMS OF DISEASE, 3935 02:39:44,800 --> 02:39:52,040 UNDERSTANDING PATHWAYS THAT ARE 3936 02:39:52,040 --> 02:39:53,800 ACTIVE IN THE NEURODEGENERATIVE 3937 02:39:53,800 --> 02:39:56,680 PROCESS AND THERAPEUTIC TARGETS 3938 02:39:56,680 --> 02:39:57,280 TO GO AFTER IN OUR DRUG 3939 02:39:57,280 --> 02:40:03,080 DEVELOPMENT. 3940 02:40:03,080 --> 02:40:07,000 TRANSLATIONAL RESEARCH IS MORE 3941 02:40:07,000 --> 02:40:11,800 FOCUSSED ON TRANSLATING OR 3942 02:40:11,800 --> 02:40:12,600 TAKING ADVANTAGE OF THOSE 3943 02:40:12,600 --> 02:40:17,680 CONVENIENCES AND DOING WHAT IS 3944 02:40:17,680 --> 02:40:23,000 NECESSARY AND THERE'S TARGET 3945 02:40:23,000 --> 02:40:23,520 VALIDATION AND INVOLVES 3946 02:40:23,520 --> 02:40:25,480 BIOMARKER DEVELOPMENT AND THESE 3947 02:40:25,480 --> 02:40:32,480 ARE BIOMARKERS OF PREDICTOR 3948 02:40:32,480 --> 02:40:43,000 PURPOSES TO TRY TO AND YOU HEARD 3949 02:40:48,760 --> 02:40:52,000 ALS IS A VERY HETEROGENEOUS 3950 02:40:52,000 --> 02:40:53,200 DISORDER AND ONE QUESTION IS 3951 02:40:53,200 --> 02:40:55,400 WHETHER THERE'S BIOMARKERS THAT 3952 02:40:55,400 --> 02:40:57,840 CAN IDENTIFY SUB TYPES THAT MAY 3953 02:40:57,840 --> 02:40:59,440 RESPOND TO ONE THSHT APPROACH 3954 02:40:59,440 --> 02:41:00,000 VERSUS ANOTHER. 3955 02:41:00,000 --> 02:41:10,520 ALL THIS WOULD HAPPEN WITHIN AN 3956 02:41:17,600 --> 02:41:18,080 IT VO 3957 02:41:18,080 --> 02:41:18,720 INVOLVES THE THERAPEUTIC FOR THE 3958 02:41:18,720 --> 02:41:20,720 BENEFIT OF OUR PATIENTS AND 3959 02:41:20,720 --> 02:41:30,880 FAMILIES. 3960 02:41:40,000 --> 02:41:42,160 AND HOW COULD IT BE WE 3961 02:41:42,160 --> 02:41:43,320 UNDERSTAND TO LITTLE ABOUT ALS. 3962 02:41:43,320 --> 02:41:45,560 THE TRUTH IS AND AS WE HEARD 3963 02:41:45,560 --> 02:41:46,760 THIS MORNING, WE UNDERSTAND A 3964 02:41:46,760 --> 02:41:49,480 LOT ABOUT ALS. 3965 02:41:49,480 --> 02:41:52,480 MUCH MORE THAN WE DID CERTAINLY 3966 02:41:52,480 --> 02:41:54,480 80 YEARS AGO AND EVEN 10, 20 3967 02:41:54,480 --> 02:41:54,880 YEARS AGO. 3968 02:41:54,880 --> 02:41:57,520 I THINK MUCH OF THE RESEARCH 3969 02:41:57,520 --> 02:41:58,720 ADVANCES WE HEARD THIS MORNING 3970 02:41:58,720 --> 02:42:01,360 COMES FROM STUDY OF THE RARE 3971 02:42:01,360 --> 02:42:02,760 GENETIC FORMS OF THE DISEASE. 3972 02:42:02,760 --> 02:42:04,720 A BETTER UNDERSTANDING OF THE 3973 02:42:04,720 --> 02:42:06,640 GENETIC LANDSCAPE OF ALS. 3974 02:42:06,640 --> 02:42:08,720 THIS HAS PROVIDED IMPORTANT 3975 02:42:08,720 --> 02:42:10,280 INSIGHT INTO DISEASE MECHANISMS 3976 02:42:10,280 --> 02:42:16,000 AND LOOK AT CELLULAR MODELS OF 3977 02:42:16,000 --> 02:42:20,520 DISEASE AND USED FOR FURTHER 3978 02:42:20,520 --> 02:42:24,720 McN 3979 02:42:24,720 --> 02:42:28,040 MECHANISTIC OF THE DISEASE AND 3980 02:42:28,040 --> 02:42:31,280 WHAT ABOUT THE OTHER 85%, 90% OF 3981 02:42:31,280 --> 02:42:33,880 PATIENTS THE GENETIC STUDIES 3982 02:42:33,880 --> 02:42:34,920 IGNORE SOME PEOPLE. 3983 02:42:34,920 --> 02:42:36,440 I ARGUE THAT'S NOT THE CASE AND 3984 02:42:36,440 --> 02:42:38,440 THE INSIGHTS GAINED ABOUT THE 3985 02:42:38,440 --> 02:42:41,080 BIOLOGY OF ALS THROUGH GENETIC 3986 02:42:41,080 --> 02:42:43,200 FORMS OF DISEASE IS HIGHLY 3987 02:42:43,200 --> 02:42:46,880 RELEVANT TO THE SPORADIC FORMS 3988 02:42:46,880 --> 02:42:48,440 OF THE DISEASE AND WE CAN APPLY 3989 02:42:48,440 --> 02:42:51,920 THE LESSONS LEARNED FROM THE 3990 02:42:51,920 --> 02:43:02,440 RARE GENETIC FORMS TO UNDERSTAND 3991 02:43:07,120 --> 02:43:08,000 SPORADIC DISEASE AND WILL DEPEND 3992 02:43:08,000 --> 02:43:12,720 ON THE EFFORT THAT EXISTS FOR 3993 02:43:12,720 --> 02:43:22,040 THE NEED OF GROWTH. 3994 02:43:22,040 --> 02:43:24,720 SO TRANSLATIONAL RESEARCH REALLY 3995 02:43:24,720 --> 02:43:27,120 INVOLVES THE MOST RELEVANT OF 3996 02:43:27,120 --> 02:43:30,080 MODELS FOR ALS, THE HUMAN MODEL. 3997 02:43:30,080 --> 02:43:33,640 WHAT WE NEED TO DO THROUGH OUR 3998 02:43:33,640 --> 02:43:38,800 TRANSLATIONAL EFFORTS IS TO 3999 02:43:38,800 --> 02:43:40,520 CHARACTERIZE OUR PATIENTS 4000 02:43:40,520 --> 02:43:44,560 THROUGH CLINICAL, 4001 02:43:44,560 --> 02:43:46,840 ELECTROPHYSIOLOGICAL MARKERS AND 4002 02:43:46,840 --> 02:43:48,640 STUDY ALL THE VARIETIES. 4003 02:43:48,640 --> 02:43:50,200 WE NEED TO DO THAT THROUGH 4004 02:43:50,200 --> 02:43:53,280 SAMPLE COLLECTION, STORAGE, 4005 02:43:53,280 --> 02:43:56,280 SHARING, USE OF BIOFLUIDS AND 4006 02:43:56,280 --> 02:43:58,920 CELL LINES YOU HEARD DISCUSSED 4007 02:43:58,920 --> 02:44:02,600 THIS MORNING AND SKIN CELLS AND 4008 02:44:02,600 --> 02:44:03,000 FIB 4009 02:44:03,000 --> 02:44:08,600 FIBROBLASTS AND PLURIPOTENT STEM 4010 02:44:08,600 --> 02:44:10,560 CELLS AND STUDY OF THE HUMAN 4011 02:44:10,560 --> 02:44:10,760 MODEL. 4012 02:44:10,760 --> 02:44:13,440 AND INFRASTRUCTURE IS NEEDED FOR 4013 02:44:13,440 --> 02:44:15,280 BIOMARKER AND DISCOVERIES AS 4014 02:44:15,280 --> 02:44:17,040 DISCUSSED AND ALL THE EFFORT 4015 02:44:17,040 --> 02:44:18,040 GENERATES A LOT OF DATA. 4016 02:44:18,040 --> 02:44:20,680 DATA THAT NEEDS TO BE CURATED, 4017 02:44:20,680 --> 02:44:23,480 HARMONIZED, STORED, MANAGED AND 4018 02:44:23,480 --> 02:44:24,680 IMPORTANTLY SHARED. 4019 02:44:24,680 --> 02:44:32,720 SAMPLES AND DATA NEED TO BE 4020 02:44:32,720 --> 02:44:43,240 SHARED BROADLY AND YOU'LL HEAR 4021 02:44:49,920 --> 02:44:53,440 THEMES MENTIONED THROUGHOUT OUR 4022 02:44:53,440 --> 02:44:54,320 DISCUSSION TODAY AND IN THE 4023 02:44:54,320 --> 02:44:58,080 DOCUMENT WE SUBMITTED TO THE 4024 02:44:58,080 --> 02:44:58,360 NIH. 4025 02:44:58,360 --> 02:45:00,400 THOSE THEMES INCLUDING TO 4026 02:45:00,400 --> 02:45:01,160 ACCELERATE TRANSLATION OF 4027 02:45:01,160 --> 02:45:05,920 FUNDAMENTAL RESEARCH INTO 4028 02:45:05,920 --> 02:45:08,320 EFFECTIVE THERAPIES, WHAT WE 4029 02:45:08,320 --> 02:45:09,720 NEED IS SUSTAINED LONG-TERM 4030 02:45:09,720 --> 02:45:12,720 INVESTMENT BY THE NIH IN 4031 02:45:12,720 --> 02:45:14,680 DEVELOPING AND MAINTAINING A 4032 02:45:14,680 --> 02:45:15,200 TRANSLATIONAL RESEARCH 4033 02:45:15,200 --> 02:45:16,960 INFRASTRUCTURE TO ENSURE 4034 02:45:16,960 --> 02:45:18,280 RESOURCES AND INFORMATION ARE 4035 02:45:18,280 --> 02:45:20,200 RETAINED AT SCALE AND ENABLE 4036 02:45:20,200 --> 02:45:23,240 MULTIPLE STAKEHOLDERS 4037 02:45:23,240 --> 02:45:25,040 REPRESENTED I THINK BEAUTIFULLY 4038 02:45:25,040 --> 02:45:31,880 IB -- IN OUR COMMITTEE TO 4039 02:45:31,880 --> 02:45:32,720 SUCCEED IN THIS SPACE. 4040 02:45:32,720 --> 02:45:34,800 IN THINK ABOUT THE CHALLENGES 4041 02:45:34,800 --> 02:45:39,520 THERE'S NO NEED TO REINVENT THE 4042 02:45:39,520 --> 02:45:40,040 WHEEL. 4043 02:45:40,040 --> 02:45:43,320 THERE'S MANY EXCELLENT 4044 02:45:43,320 --> 02:45:44,560 TRANSLATIONAL EFFORTS SPONSORED 4045 02:45:44,560 --> 02:45:49,720 BY NIH THE DOD AS DR. KOROSHETZ 4046 02:45:49,720 --> 02:45:52,040 MENTIONED THIS MORNING, IN 4047 02:45:52,040 --> 02:45:52,640 RELATED DISORDER LIKE 4048 02:45:52,640 --> 02:45:56,680 ALZHEIMER'S AND PARKINSON'S 4049 02:45:56,680 --> 02:45:58,040 DISEASE AND FRONTAL TEMPORAL 4050 02:45:58,040 --> 02:45:58,320 DEMENTIA. 4051 02:45:58,320 --> 02:46:02,520 WE DON'T NEED TO REINVENT THE 4052 02:46:02,520 --> 02:46:03,040 WHEEL. 4053 02:46:03,040 --> 02:46:05,760 WE NEED TO BUILD ON THE MODELS 4054 02:46:05,760 --> 02:46:08,120 AND ON ALS AND INCORPORATE 4055 02:46:08,120 --> 02:46:10,080 EXISTING EFFORTS ONGOING WITHIN 4056 02:46:10,080 --> 02:46:12,720 THE ALS RESEARCH COMMUNITY SOME 4057 02:46:12,720 --> 02:46:14,000 OF THAT YOU HEARD ABOUT THIS 4058 02:46:14,000 --> 02:46:16,040 MORNING AND TARGETED ALS AND 4059 02:46:16,040 --> 02:46:20,360 PROJECT ALS. 4060 02:46:20,360 --> 02:46:26,440 THERE'S MANY GROUPS SUPPORTING 4061 02:46:26,440 --> 02:46:27,600 INITIATIVES AND WE NEED THE 4062 02:46:27,600 --> 02:46:29,400 STRUCTURE TO DO THIS BIGGER AND 4063 02:46:29,400 --> 02:46:29,640 BETTER. 4064 02:46:29,640 --> 02:46:34,840 WE NEED TO WORK ACROSS DISEASES 4065 02:46:34,840 --> 02:46:40,680 TO INTEGRATE DATA FROM MULTIPLE 4066 02:46:40,680 --> 02:46:44,120 DISORDER AND TO BREAK DOWN THE 4067 02:46:44,120 --> 02:46:46,120 SILOS THAT EXIST BETWEEN THE 4068 02:46:46,120 --> 02:46:48,720 DISORDERS IMPEDING OUR PROGRESS 4069 02:46:48,720 --> 02:46:58,840 IN ALS. 4070 02:47:02,200 --> 02:47:12,480 KEY TO OUR SUCCESS IS 4071 02:47:15,200 --> 02:47:19,520 PUBLIC-PRIVATE PARTNERSHIPS TO 4072 02:47:19,520 --> 02:47:22,080 BENEFIT OUR PATIENTS. 4073 02:47:22,080 --> 02:47:24,600 THE NIH HAS AN IMPORTANT ROLE TO 4074 02:47:24,600 --> 02:47:28,000 PLAY IN FACILITATING THIS 4075 02:47:28,000 --> 02:47:30,000 BETWEEN INDUSTRY AND ACADEMIA 4076 02:47:30,000 --> 02:47:35,640 AND ENCOURAGING PUBLIC RESOURCES 4077 02:47:35,640 --> 02:47:39,000 AND INDUSTRY NEEDS TO SHARE DATA 4078 02:47:39,000 --> 02:47:41,640 FROM TRIALS TO THE BROAD ALS 4079 02:47:41,640 --> 02:47:42,840 RESEARCH COMMUNITY TO ADVANCE 4080 02:47:42,840 --> 02:47:43,400 OUR COMMON GOALS. 4081 02:47:43,400 --> 02:47:49,240 SO WITH THAT, I'D LIKE TO TURN 4082 02:47:49,240 --> 02:47:52,480 IT OVER TO STACIE TO REVIEW OUR 4083 02:47:52,480 --> 02:47:53,520 SPECIFIC PRIORITY 4084 02:47:53,520 --> 02:47:53,920 RECOMMENDATIONS. 4085 02:47:53,920 --> 02:47:54,480 >>THANK YOU. 4086 02:47:54,480 --> 02:47:56,720 I'LL TRY TO KEEP THIS BRIEF TO 4087 02:47:56,720 --> 02:47:58,120 HAVE MORE TIME FOR QUESTIONS AND 4088 02:47:58,120 --> 02:48:08,320 DISCUSSIONS. 4089 02:48:12,160 --> 02:48:13,960 WE'LL BUILD ON THESE IN A LITTLE 4090 02:48:13,960 --> 02:48:18,280 BIT OF DETAIL BUT WAYS THE NIH 4091 02:48:18,280 --> 02:48:21,680 CAN CONTRIBUTE TO THE GREATER 4092 02:48:21,680 --> 02:48:26,800 EFFORTS OF TRYING TO TRANSLATE 4093 02:48:26,800 --> 02:48:29,440 THE RESEARCH FOR THESE DISEASE 4094 02:48:29,440 --> 02:48:31,960 AND TO ESTABLISH A NETWORK OF 4095 02:48:31,960 --> 02:48:33,920 ALS CENTERS OF EXCELLENCE TO 4096 02:48:33,920 --> 02:48:39,720 SUPPORT ALS TRANSLATIONAL 4097 02:48:39,720 --> 02:48:41,600 RESEARCH. 4098 02:48:41,600 --> 02:48:43,360 AND THE DESIGN IS NOT TO FOCUS 4099 02:48:43,360 --> 02:48:46,080 ON THE CARE BUT THE GOAL IS TO 4100 02:48:46,080 --> 02:48:47,880 HELP US UNDERSTAND THE DISEASE 4101 02:48:47,880 --> 02:48:48,680 BETTER AND SUPPORT 4102 02:48:48,680 --> 02:48:50,240 INFRASTRUCTURE SO THAT WE CAN 4103 02:48:50,240 --> 02:48:52,080 DEVELOP THERAPEUTICS AND DEVELOP 4104 02:48:52,080 --> 02:48:54,720 BETTER STANDARDS OF CARE THAT 4105 02:48:54,720 --> 02:48:58,080 CAN THEN BE SHARED ACROSS THE 4106 02:48:58,080 --> 02:49:00,120 GREATER COMMUNITY TO HOSPITALS 4107 02:49:00,120 --> 02:49:00,920 AND PHYSICIANS EVERYWHERE. 4108 02:49:00,920 --> 02:49:02,800 BUT BY FOCUSSING ON THE CENTERS 4109 02:49:02,800 --> 02:49:05,640 OF EXCELLENCE WE'RE GOING TO BE 4110 02:49:05,640 --> 02:49:08,240 ABLE TO LEVERAGE A LOT OF THE 4111 02:49:08,240 --> 02:49:09,680 GREAT RESEARCH BEING DONE ACROSS 4112 02:49:09,680 --> 02:49:10,360 DIFFERENT DISEASE AREAS TO LEARN 4113 02:49:10,360 --> 02:49:14,600 FROM THOSE AND TO BE ABLE TO 4114 02:49:14,600 --> 02:49:17,200 REALLY TAKE BASIC SCIENCE 4115 02:49:17,200 --> 02:49:18,880 DISCOVERIES AND BE ABLE TO 4116 02:49:18,880 --> 02:49:20,840 SUPPORT MORE BASIC SCIENCE 4117 02:49:20,840 --> 02:49:23,680 RESEARCH THROUGH THE LARGE DATA 4118 02:49:23,680 --> 02:49:25,520 REPOSITORIES THAT BY DEFINITION 4119 02:49:25,520 --> 02:49:29,400 NEED TO BE CONCENTRATED FOR 4120 02:49:29,400 --> 02:49:30,520 EFFICIENCY AND CONSISTENCY IN 4121 02:49:30,520 --> 02:49:32,120 HOW SAMPLES ARE COLLECTED AND 4122 02:49:32,120 --> 02:49:33,960 STORED AND USED. 4123 02:49:33,960 --> 02:49:36,680 AND IN SHARING THAT DATA BROADLY 4124 02:49:36,680 --> 02:49:38,960 WITH THE ENTIRE COMMUNITY OF 4125 02:49:38,960 --> 02:49:42,360 RESEARCHERS SO THAT WE CAN 4126 02:49:42,360 --> 02:49:43,800 TRANSLATE FINDINGS OUT OF THE 4127 02:49:43,800 --> 02:49:45,000 CENTERS OF EXCELLENCE INTO 4128 02:49:45,000 --> 02:49:45,520 BETTER CARE AND BETTER 4129 02:49:45,520 --> 02:49:48,640 THERAPEUTICS. 4130 02:49:48,640 --> 02:49:49,640 SO WITHIN THESE CENTERS OF 4131 02:49:49,640 --> 02:49:51,560 EXCELLENCE WE'RE PROPOSING A 4132 02:49:51,560 --> 02:49:54,080 PROGRAM PROJECT CORE THAT REALLY 4133 02:49:54,080 --> 02:49:55,440 IS FOCUSSED ON STUDYING THE 4134 02:49:55,440 --> 02:49:58,120 BASIC MECHANISMS OF ALS AND 4135 02:49:58,120 --> 02:49:59,200 TRYING TO IDENTIFY NEW 4136 02:49:59,200 --> 02:50:01,120 THERAPEUTIC TARGETS. 4137 02:50:01,120 --> 02:50:05,800 THERE'LL BE A CLINICAL CORE TO 4138 02:50:05,800 --> 02:50:07,920 OVERSEE BIO FLUID COLLECTION AND 4139 02:50:07,920 --> 02:50:09,960 DEEP PHENOTYPING AND THAT'S 4140 02:50:09,960 --> 02:50:10,640 INCREDIBLY IMPORTANT AND A WANT 4141 02:50:10,640 --> 02:50:14,760 TO EMPHASIZE JUST COLLECTING BIO 4142 02:50:14,760 --> 02:50:18,840 FLUIDS AND SAMPLES FROM PATIENTS 4143 02:50:18,840 --> 02:50:22,200 AND JUST CATEGORIZING AS ALS IS 4144 02:50:22,200 --> 02:50:24,600 NOT ENOUGH WE NEED DEEP 4145 02:50:24,600 --> 02:50:25,480 PHENOTYPING AND UNDERSTANDING 4146 02:50:25,480 --> 02:50:27,000 THAT GOES BEHIND EACH SAMPLE 4147 02:50:27,000 --> 02:50:29,040 WHICH IS HOW FAST DOES THE 4148 02:50:29,040 --> 02:50:29,960 DISEASE PROGRESS. 4149 02:50:29,960 --> 02:50:31,760 WHAT'S THE GENETIC BEHIND THE 4150 02:50:31,760 --> 02:50:32,560 DISEASE? 4151 02:50:32,560 --> 02:50:34,400 HOW DO WE PAIR THE DIFFERENT 4152 02:50:34,400 --> 02:50:36,200 BIOMARKERS WE SEE WITH THE 4153 02:50:36,200 --> 02:50:37,800 CLINICAL PICTURE? 4154 02:50:37,800 --> 02:50:40,320 BECAUSE I THINK WE UNDERSTAND 4155 02:50:40,320 --> 02:50:42,680 THAT ALS CONSISTS OF A GROUP OF 4156 02:50:42,680 --> 02:50:43,720 DISEASES THAT CAN ACTUALLY BE 4157 02:50:43,720 --> 02:50:45,960 CAUSED BY MUTATIONS IN DIFFERENT 4158 02:50:45,960 --> 02:50:48,080 GENES OR DIFFERENT RISK FACTORS 4159 02:50:48,080 --> 02:50:51,600 AND BEING ABLE TO UNDERSTAND 4160 02:50:51,600 --> 02:50:57,240 IT'S NOT ALS IS ALS IS ALS JUST 4161 02:50:57,240 --> 02:50:58,440 LIKE YOU DON'T SAY SOMEONE WITH 4162 02:50:58,440 --> 02:51:00,000 CANCER IS ALL THE SAME WE HAVE 4163 02:51:00,000 --> 02:51:04,560 TO UNDERSTAND THE GENETICS 4164 02:51:04,560 --> 02:51:08,080 DERIVING THAT FORM OF CANCER TO 4165 02:51:08,080 --> 02:51:10,080 PAIR IT WITH THE RIGHT 4166 02:51:10,080 --> 02:51:12,120 THERAPEUTIC SO WE NEED DEEP 4167 02:51:12,120 --> 02:51:13,720 CLINICAL PHENOTYPING TO 4168 02:51:13,720 --> 02:51:14,720 UNDERSTAND WHAT AN INDIVIDUAL 4169 02:51:14,720 --> 02:51:18,000 ALS LOOKS LIKE TO UNDERSTAND THE 4170 02:51:18,000 --> 02:51:19,280 DIFFERENCES BETWEEN PATIENTS SO 4171 02:51:19,280 --> 02:51:20,560 EVENTUALLY WE'LL BE ABLE TO PAIR 4172 02:51:20,560 --> 02:51:21,680 THE RIGHT PATIENTS TO THE RIGHT 4173 02:51:21,680 --> 02:51:32,040 TYPES OF THERAPEUTICS. 4174 02:51:33,040 --> 02:51:34,080 WE PROVIDE CHARACTERIZATION OF 4175 02:51:34,080 --> 02:51:36,680 ALL PARTICIPANTS TO UNDERSTAND 4176 02:51:36,680 --> 02:51:38,360 THE GENETICS TO GO WITH THE 4177 02:51:38,360 --> 02:51:42,960 CLINICAL PHENOTYPING TO BE ABLE 4178 02:51:42,960 --> 02:51:46,120 TO SUBDIVIDE PATIENTS TO 4179 02:51:46,120 --> 02:51:47,760 UNDERSTAND THE DISEASE 4180 02:51:47,760 --> 02:51:49,920 PROGRESSION AND HOW IT'S 4181 02:51:49,920 --> 02:51:51,000 DIFFERENT ACROSS GENETIC 4182 02:51:51,000 --> 02:51:52,080 DRIVERS. 4183 02:51:52,080 --> 02:51:59,280 AND WE NEED AN ADMINISTRATIVE 4184 02:51:59,280 --> 02:52:00,720 CORE TO PROVIDE ASSISTANCE 4185 02:52:00,720 --> 02:52:02,920 THROUGHOUT THE PROGRAM. 4186 02:52:02,920 --> 02:52:07,840 SO BUILDING ON THE IMPORTANCE OF 4187 02:52:07,840 --> 02:52:10,040 THE BIO SAMPLES AND DATA 4188 02:52:10,040 --> 02:52:10,640 INFRASTRUCTURE, IDENTIFICATION 4189 02:52:10,640 --> 02:52:14,280 OF MARKERS AND MECHANISMS THAT 4190 02:52:14,280 --> 02:52:15,920 DISTINGUISH THE SUBTYPES OF ALS 4191 02:52:15,920 --> 02:52:16,680 IS SO IMPORTANT. 4192 02:52:16,680 --> 02:52:20,960 AGAIN, I THINK WE NEED TO 4193 02:52:20,960 --> 02:52:23,920 UNDERSTAND WHAT AN INDIVIDUAL'S 4194 02:52:23,920 --> 02:52:26,440 ALS IS BEING DRIVEN BY 4195 02:52:26,440 --> 02:52:27,840 BIOLOGICALLY TO PAIR WITH THE 4196 02:52:27,840 --> 02:52:28,400 RIGHT TREATMENT. 4197 02:52:28,400 --> 02:52:31,560 TO DO THAT REQUIRES LOOKING AT A 4198 02:52:31,560 --> 02:52:33,240 LOT OF PATIENTS WITH ALS HAVING 4199 02:52:33,240 --> 02:52:35,200 BEEN DEEPLY PHENOTYPED AND 4200 02:52:35,200 --> 02:52:37,480 STUDIED TO LOOK FOR BIOMARKERS 4201 02:52:37,480 --> 02:52:37,960 OF DISEASE. 4202 02:52:37,960 --> 02:52:40,000 WE HEARD ABOUT THE IMPORTANCE OF 4203 02:52:40,000 --> 02:52:40,280 BIOMARKERS. 4204 02:52:40,280 --> 02:52:43,720 WE'LL HEAR MORE IN THIS SECTION, 4205 02:52:43,720 --> 02:52:46,080 BIOMARKERS, BIOMARKERS, 4206 02:52:46,080 --> 02:52:48,360 BIOMARKERS NOT BE EMPHASIZED 4207 02:52:48,360 --> 02:52:49,560 ENOUGH. 4208 02:52:49,560 --> 02:52:56,160 WE ALL UNDERSTAND THAT TO TREAT 4209 02:52:56,160 --> 02:52:57,800 ANY NEURODEGENERATIVE DISEASE, 4210 02:52:57,800 --> 02:52:59,520 ALS INCLUDED, AS EARLY AS 4211 02:52:59,520 --> 02:53:01,280 POSSIBLE WE'LL WANT TO TREAT 4212 02:53:01,280 --> 02:53:04,160 BEFORE SOMEONE IS SHOWING 4213 02:53:04,160 --> 02:53:06,200 SYMPTOMS OR AT LEAST AS EARLY AS 4214 02:53:06,200 --> 02:53:06,600 IS POSSIBLE. 4215 02:53:06,600 --> 02:53:09,440 HOW WILL THAT BE ACHIEVED? 4216 02:53:09,440 --> 02:53:10,000 BIOMARKERS. 4217 02:53:10,000 --> 02:53:11,960 WE NEED TO UNDERSTAND WHEN THE 4218 02:53:11,960 --> 02:53:14,120 BIOMARKERS START TO MUTATE AND 4219 02:53:14,120 --> 02:53:19,240 USE THEM TO SUPPORT CLINICAL 4220 02:53:19,240 --> 02:53:20,000 TRIALS. 4221 02:53:20,000 --> 02:53:25,280 I CAN'T OVERSTATE THE IMPORTANCE 4222 02:53:25,280 --> 02:53:28,040 OF BIOMARKERS. 4223 02:53:28,040 --> 02:53:31,720 TO BE SUCCESSFUL WE'LL NEED A 4224 02:53:31,720 --> 02:53:34,080 LARGE SCALE STANDARDIZED 4225 02:53:34,080 --> 02:53:35,080 REPOSITORY AND HENRIK WILL SPEAK 4226 02:53:35,080 --> 02:53:36,320 MORE TO THE IMPORTANCE OF HOW 4227 02:53:36,320 --> 02:53:39,720 WE'RE COLLECTING THE BIOMARKERS 4228 02:53:39,720 --> 02:53:41,320 KEPT IN THE FOREFRONT OF 4229 02:53:41,320 --> 02:53:42,480 STANDARDIZATION AND WE NEED TO 4230 02:53:42,480 --> 02:53:43,920 COLLECT THEM IN THE SAME WAY AND 4231 02:53:43,920 --> 02:53:46,280 BEING TREATED IN THE SAME WAY SO 4232 02:53:46,280 --> 02:53:51,080 WE CAN COMPARE ACROSS STUDIES. 4233 02:53:51,080 --> 02:53:52,080 IT IS ALSO INCREDIBLY IMPORTANT 4234 02:53:52,080 --> 02:53:54,120 THE SAMPLES BE BROADLY AVAILABLE 4235 02:53:54,120 --> 02:53:56,560 TO THOSE SCIENTISTS IN ACADEMIA 4236 02:53:56,560 --> 02:53:57,240 AS WELL AS INDUSTRY. 4237 02:53:57,240 --> 02:54:00,240 WHEN WE'RE TALKING ABOUT 4238 02:54:00,240 --> 02:54:02,600 DEVELOPING NEW THERAPEUTICS 4239 02:54:02,600 --> 02:54:04,960 THAT'S GOING TO COME FROM NOVEL 4240 02:54:04,960 --> 02:54:06,960 DISCOVERIES IN ACADEMIA AND 4241 02:54:06,960 --> 02:54:10,360 TRANSLATION OF DISCOVERY AND 4242 02:54:10,360 --> 02:54:12,600 INDUSTRY THAT REQUIRES US TO 4243 02:54:12,600 --> 02:54:14,200 BOTH SUPPORT THE INDUSTRIAL 4244 02:54:14,200 --> 02:54:16,680 SCIENTISTS TRYING TO DEVELOP THE 4245 02:54:16,680 --> 02:54:17,160 THERAPEUTICS. 4246 02:54:17,160 --> 02:54:22,840 AGAIN, WE DON'T NEED TO REINVENT 4247 02:54:22,840 --> 02:54:33,240 THE WHEEL AND FINALLY A 4248 02:54:37,520 --> 02:54:44,280 SIGNIFICANT PRIORITY AND THERE'S 4249 02:54:44,280 --> 02:54:54,920 A NOVEL APPROACH WHETHER 4250 02:54:54,920 --> 02:54:56,680 KNOCKING DOWN A DREAM AND AWAY 4251 02:54:56,680 --> 02:54:59,280 HEARD ABOUT CRISPR AND GENE 4252 02:54:59,280 --> 02:54:59,520 EDITING. 4253 02:54:59,520 --> 02:55:00,040 THERE'S INCREDIBLE NEW 4254 02:55:00,040 --> 02:55:02,080 TECHNOLOGIES AT OUR FINGER TIPS 4255 02:55:02,080 --> 02:55:04,920 BUT THERE'S STILL A LOT OF 4256 02:55:04,920 --> 02:55:08,720 DEVELOPMENT TO GO INTO ACTUALLY 4257 02:55:08,720 --> 02:55:15,600 HAVING THESE READY FOR USE IN 4258 02:55:15,600 --> 02:55:16,560 THESE POPULATIONS. 4259 02:55:16,560 --> 02:55:18,960 SO HAVING FEDERAL SUPPORT TO 4260 02:55:18,960 --> 02:55:20,640 VALIDATE THE NOVEL TARGETS AS 4261 02:55:20,640 --> 02:55:23,000 WELL NOVEL TECHNOLOGIES IS GOING 4262 02:55:23,000 --> 02:55:28,080 TO BE INCREDIBLY IMPORTANT AND A 4263 02:55:28,080 --> 02:55:28,400 WILLINGNESS. 4264 02:55:28,400 --> 02:55:29,560 THERE'S RISK INVOLVED HERE TO 4265 02:55:29,560 --> 02:55:30,360 BRING THE NOVEL TECHNOLOGIES 4266 02:55:30,360 --> 02:55:34,080 FORWARD AND WE NEED TO 4267 02:55:34,080 --> 02:55:37,360 UNDERSTAND THAT AND WAYS TO DO 4268 02:55:37,360 --> 02:55:39,000 EXPLORATORY TRIALS. 4269 02:55:39,000 --> 02:55:43,240 WE ALSO ENCOURAGE MORE 4270 02:55:43,240 --> 02:55:44,360 PUBLIC-PRIVATE PARTNERSHIP AND 4271 02:55:44,360 --> 02:55:46,080 ACADEMIA AND INDUSTRY BECAUSE IT 4272 02:55:46,080 --> 02:55:52,080 WILL TAKE THE COLLABORATION TO 4273 02:55:52,080 --> 02:55:54,720 TAKE THESE TO THERAPEUTICS. 4274 02:55:54,720 --> 02:55:56,680 WE ALSO THINK HAVING THE SUPPORT 4275 02:55:56,680 --> 02:55:58,680 THROUGH THE ALS CENTERS OF 4276 02:55:58,680 --> 02:55:59,800 EXCELLENCE AND THROUGH THE CORPS 4277 02:55:59,800 --> 02:56:03,880 WE TALK ABOUT TO ENABLE CLINICAL 4278 02:56:03,880 --> 02:56:06,600 TRIALS WHETHER THAT THE BE 4279 02:56:06,600 --> 02:56:08,680 PERFORMED BEEN THE CENTERS OF 4280 02:56:08,680 --> 02:56:10,440 EXCELLENCE OR CLINICAL END 4281 02:56:10,440 --> 02:56:12,720 POINTS OR DEVELOPED IN 4282 02:56:12,720 --> 02:56:14,000 COLLABORATION IN THE BIOMARKER 4283 02:56:14,000 --> 02:56:15,600 CORE, ALL THAT WILL BE IMPORTANT 4284 02:56:15,600 --> 02:56:18,080 TO ENABLE EFFICIENT TRIALS BASED 4285 02:56:18,080 --> 02:56:19,960 ON BIOMARKERS AND ENROLL THE 4286 02:56:19,960 --> 02:56:22,080 RIGHT PATIENTS AT THE RIGHT TIME 4287 02:56:22,080 --> 02:56:23,960 AND QUICKLY SEE IF WE'RE HAVING 4288 02:56:23,960 --> 02:56:26,800 AN EFFECT ON THESE. 4289 02:56:26,800 --> 02:56:32,680 AND WITH THAT I'LL PASS IT OVER 4290 02:56:32,680 --> 02:56:41,280 TO YOU, JOE, TO TALK MORE. 4291 02:56:41,280 --> 02:56:42,920 THANK YOU, STACIE AND NEIL 4292 02:56:42,920 --> 02:56:43,760 OUTLINING THE PRIORITIES OUR 4293 02:56:43,760 --> 02:56:50,360 TEAM HAS COME UP WITH. 4294 02:56:50,360 --> 02:56:53,080 I'LL TRY TO DO LEVEL SETTING 4295 02:56:53,080 --> 02:56:54,720 REGARDING WHAT'S INVOLVED IN THE 4296 02:56:54,720 --> 02:56:58,040 DRUG DISCOVERY PROCESS AND THEN 4297 02:56:58,040 --> 02:56:59,760 OUTLINE HOW THE PRIORITIES THAT 4298 02:56:59,760 --> 02:57:01,960 WERE GENERATED BY OUR TEAM CAN 4299 02:57:01,960 --> 02:57:02,960 IMPACT THIS DRUG DISCOVERY 4300 02:57:02,960 --> 02:57:10,600 PROCESS IN A POSITIVE WAY. 4301 02:57:10,600 --> 02:57:21,120 AND TO START AND DRUG DISCOVERY 4302 02:57:39,240 --> 02:57:40,240 CANDIDATES DEFINITELY APPLY AND 4303 02:57:40,240 --> 02:57:43,160 AS I THINK MOST FOLKS ON THE 4304 02:57:43,160 --> 02:57:45,160 CALL ARE AWARE IN ALS THE 4305 02:57:45,160 --> 02:57:46,480 CHALLENGES ARE EVEN GREATER THAN 4306 02:57:46,480 --> 02:57:49,480 I WOULD SAY ON AVERAGE FOR ANY 4307 02:57:49,480 --> 02:57:51,520 DRUG DEVELOPMENT CANDIDATE. 4308 02:57:51,520 --> 02:57:55,360 SO JUST TO POINT OUT A FEW 4309 02:57:55,360 --> 02:57:56,760 THINGS IN THIS FUNNEL CHART ON 4310 02:57:56,760 --> 02:58:04,560 THE LEFT, I THINK MOST PEOPLE 4311 02:58:04,560 --> 02:58:06,720 AND EARLY STAGE CLINICAL TRIALS 4312 02:58:06,720 --> 02:58:09,000 ARE GOING THROUGH PHASE 3 TRIALS 4313 02:58:09,000 --> 02:58:10,080 AND WHAT IS PERHAPS LESS 4314 02:58:10,080 --> 02:58:11,880 FAMILIAR ARE ALL THE STEPS THAT 4315 02:58:11,880 --> 02:58:16,040 LEAD UP TO THOSE CLINICAL 4316 02:58:16,040 --> 02:58:16,280 TRIALS. 4317 02:58:16,280 --> 02:58:20,320 THEY INVOLVE SCREENING A LARGE 4318 02:58:20,320 --> 02:58:21,880 NUMBER OF POTENTIAL THERAPEUTICS 4319 02:58:21,880 --> 02:58:22,680 CANDIDATES IN ORDER TO IDENTIFY 4320 02:58:22,680 --> 02:58:26,120 WHAT ARE A SMALL NUMBER OF 4321 02:58:26,120 --> 02:58:27,720 COMPOUNDS THAT ARE SUFFICIENTLY 4322 02:58:27,720 --> 02:58:29,400 ACTIVE AND SAFE TO ENTER 4323 02:58:29,400 --> 02:58:29,920 CLINICAL TRIALS. 4324 02:58:29,920 --> 02:58:33,160 AND I THINK THIS HIGHLIGHTS THE 4325 02:58:33,160 --> 02:58:35,880 NUMBERS INVOLVED AS WELL AS THE 4326 02:58:35,880 --> 02:58:42,040 TIME AND COST AND ALSO KNOWING 4327 02:58:42,040 --> 02:58:45,440 THERE'S MANY FAILURES AS STACIE 4328 02:58:45,440 --> 02:58:47,880 MENTIONED AND IF WE WANT A HIGH 4329 02:58:47,880 --> 02:58:49,720 LEVEL OF SUCCESS WE NEED A HIGH 4330 02:58:49,720 --> 02:58:54,800 LEVEL OF INVESTMENT. 4331 02:58:54,800 --> 02:59:05,320 AND ON AVERAGE AND SO I THINK 4332 02:59:08,000 --> 02:59:09,000 THIS HIGHLIGHTS THEN SOME OF THE 4333 02:59:09,000 --> 02:59:13,680 IDEAS THAT OUR WORK GROUP CAME 4334 02:59:13,680 --> 02:59:19,640 UP WITH THE TIME AND NUMBERS 4335 02:59:19,640 --> 02:59:21,040 INVOLVED HOW DO YOU OPTIMIZE HOW 4336 02:59:21,040 --> 02:59:24,040 THE FUNNEL WORKS AND GIVE DRUG 4337 02:59:24,040 --> 02:59:31,040 CANDIDATES THE BEST CHANCE OF 4338 02:59:31,040 --> 02:59:35,240 SUCCESS. 4339 02:59:35,240 --> 02:59:37,760 AND THE ONE I'LL HIGHLIGHT IS 4340 02:59:37,760 --> 02:59:39,280 ONE STACIE MENTIONED IN PRIORITY 4341 02:59:39,280 --> 02:59:40,760 3 AROUND THE TRANSLATION BETWEEN 4342 02:59:40,760 --> 02:59:42,400 YOUR INITIATIVE DISCOVERY AND 4343 02:59:42,400 --> 02:59:47,400 THE DRUG DISCOVERY PROCESS. 4344 02:59:47,400 --> 02:59:48,000 INITIATING THE CANDIDATE 4345 02:59:48,000 --> 02:59:58,720 THERAPEUTIC SPACE ON THAT IDEA. 4346 02:59:58,720 --> 03:00:00,880 AND THE FIRST BAR TENDS TO BE 4347 03:00:00,880 --> 03:00:03,240 BEST DONE BUT NOT ALWAYS IN 4348 03:00:03,240 --> 03:00:05,520 ACADEMIA AND THE BAR'S AFTER IT 4349 03:00:05,520 --> 03:00:07,400 ARE MOST OFTEN DONE IN INDUSTRY. 4350 03:00:07,400 --> 03:00:09,560 SO ANYTHING WE CAN DO TO LOWER 4351 03:00:09,560 --> 03:00:11,280 THE BAR AND MAKE THAT MORE 4352 03:00:11,280 --> 03:00:13,120 EFFECTIVE IS GOING TO HAVE A 4353 03:00:13,120 --> 03:00:15,160 HUGE IMPACT ON OVER ALL SUCCESS. 4354 03:00:15,160 --> 03:00:18,960 AND THE WAY I LIKE TO THINK 4355 03:00:18,960 --> 03:00:21,240 ABOUT IT IS AND GARBAGE IN, 4356 03:00:21,240 --> 03:00:22,800 GARBAGE OUT, IF YOU START THE 4357 03:00:22,800 --> 03:00:24,680 FUNNEL WITH NOT THE BEST IDEAS 4358 03:00:24,680 --> 03:00:26,840 YOU'LL END WITH NOT THE BEST 4359 03:00:26,840 --> 03:00:27,040 IDEAS. 4360 03:00:27,040 --> 03:00:31,240 THAT'S A PRETTY DISAPPOINTING 4361 03:00:31,240 --> 03:00:33,160 PLACE TO BE OVER A DECADE OF 4362 03:00:33,160 --> 03:00:33,360 WORK. 4363 03:00:33,360 --> 03:00:36,200 IT'S CRITICALLY IMPORTANT TO 4364 03:00:36,200 --> 03:00:38,280 MAKE SURE THE BEST SCIENTIFIC 4365 03:00:38,280 --> 03:00:44,720 IDEAS ARE MOVING FORWARD INTO 4366 03:00:44,720 --> 03:00:47,560 DRUG DISCOVERY EFFORTS. 4367 03:00:47,560 --> 03:00:51,000 AND THE SECOND POINT STACIE AND 4368 03:00:51,000 --> 03:00:52,040 NEIL HIGHLIGHTED AROUND BIO 4369 03:00:52,040 --> 03:00:52,560 SAMPLES AND THE USE OF 4370 03:00:52,560 --> 03:01:02,000 BIOMARKERS. 4371 03:01:02,000 --> 03:01:04,600 AND MAKING SURE WE TAKE THE BEST 4372 03:01:04,600 --> 03:01:09,600 IDEAS FORWARD IN DRUG DISCOVERY. 4373 03:01:09,600 --> 03:01:16,120 THE BOTTOM LINE IS OUR 4374 03:01:16,120 --> 03:01:18,360 UNDERSTANDING AND IT'S WHAT THE 4375 03:01:18,360 --> 03:01:20,360 USE OF OUR SAMPLES AT THE EARLY 4376 03:01:20,360 --> 03:01:27,680 STAGE CAN DO IS TO MAKE SURE 4377 03:01:27,680 --> 03:01:28,680 WE'RE PURSUING NOT JUST ALS AND 4378 03:01:28,680 --> 03:01:31,400 WE'VE BEEN ABLE TO DO THAT MANY 4379 03:01:31,400 --> 03:01:40,120 TIMES AND IT JUST DOESN'T HAVE 4380 03:01:40,120 --> 03:01:47,160 THE SAME IMPACT. 4381 03:01:47,160 --> 03:01:48,720 AND IN TERMS OF IDENTIFYING THE 4382 03:01:48,720 --> 03:01:51,200 RIGHT PATIENTS TO BE INVOLVED IN 4383 03:01:51,200 --> 03:01:53,960 TRIALS AND HOW TO MEASURE EARLY 4384 03:01:53,960 --> 03:01:54,600 EFFECTIVENESS OF COMPOUNDS 4385 03:01:54,600 --> 03:01:56,280 WITHIN CLINICAL TRIALS IS 4386 03:01:56,280 --> 03:02:00,000 ANOTHER KEY POINT WHERE BIO 4387 03:02:00,000 --> 03:02:01,200 SAMPLES HAVE PLAYED A KEY ROLE 4388 03:02:01,200 --> 03:02:03,680 AND WILL CONTINUE TO DO SO AND 4389 03:02:03,680 --> 03:02:06,280 HAVING LARGE EASILY ACCESSIBLE 4390 03:02:06,280 --> 03:02:07,920 REPOSITORIES OF THE SAMPLES TO 4391 03:02:07,920 --> 03:02:15,080 BE LEVERAGED BY AC DIM -- 4392 03:02:15,080 --> 03:02:18,600 ACADEMICS AND INDUSTRY ALIKE 4393 03:02:18,600 --> 03:02:19,600 WILL BE NECESSARY TO BE 4394 03:02:19,600 --> 03:02:22,120 EFFECTIVE AND ONCOLOGY IS AN 4395 03:02:22,120 --> 03:02:25,080 AREA THAT'S HAD MORE SUCCESS AND 4396 03:02:25,080 --> 03:02:26,400 THE BOTTOM LINE OR A BIG PART OF 4397 03:02:26,400 --> 03:02:28,160 THE REASON WHY IS BECAUSE YOU 4398 03:02:28,160 --> 03:02:30,600 DON'T JUST TREAT CANCER ANYMORE, 4399 03:02:30,600 --> 03:02:32,720 YOU TREAT A SPECIFIC SUBTYPE OF 4400 03:02:32,720 --> 03:02:33,680 CANCER IDENTIFIED THROUGH THE 4401 03:02:33,680 --> 03:02:35,120 SAME TYPE OF BIOMARKERS WHICH IS 4402 03:02:35,120 --> 03:02:37,240 SOMETHING THAT IS REALLY GOING 4403 03:02:37,240 --> 03:02:44,680 TO BE CRITICAL TO TRANSLATE INTO 4404 03:02:44,680 --> 03:02:49,240 ALS. 4405 03:02:49,240 --> 03:02:51,160 AND A GREAT EXAMPLE MENTIONED 4406 03:02:51,160 --> 03:02:55,720 EARLIER TODAY WE LEARNED A LOT 4407 03:02:55,720 --> 03:03:00,040 FROM SPECIFIC GENETICALLY 4408 03:03:00,040 --> 03:03:04,120 DEFINED SUB POPULATIONS OF ALS 4409 03:03:04,120 --> 03:03:05,000 PATIENTS AND BIOMARKER WILL BE 4410 03:03:05,000 --> 03:03:06,320 KEY TO EXTENDING THOSE 4411 03:03:06,320 --> 03:03:07,080 DISCOVERIES AND UNDERSTANDING 4412 03:03:07,080 --> 03:03:10,800 WHICH OF THOSE PATHWAYS MAY BE 4413 03:03:10,800 --> 03:03:11,720 RELEVANT IN THE BROADER ALS 4414 03:03:11,720 --> 03:03:12,360 POPULATION. 4415 03:03:12,360 --> 03:03:13,480 THAT'S A VERY CONCRETE EXAMPLE 4416 03:03:13,480 --> 03:03:17,360 OF HOW THESE CAN BE USED TO 4417 03:03:17,360 --> 03:03:18,520 EXTEND THE POTENTIAL OF 4418 03:03:18,520 --> 03:03:19,280 THERAPIES IN ALS. 4419 03:03:19,280 --> 03:03:21,400 I'LL STOP THERE AND HAND IT OVER 4420 03:03:21,400 --> 03:03:23,080 TO HENRIK WHO WILL TALK A LITTLE 4421 03:03:23,080 --> 03:03:24,560 BIT MORE IN DETAIL ABOUT THE ALS 4422 03:03:24,560 --> 03:03:29,440 BIOMARKERS. 4423 03:03:29,440 --> 03:03:30,560 >>THANK YOU. 4424 03:03:30,560 --> 03:03:31,960 THIS IS A SIMPLE SLIDE 4425 03:03:31,960 --> 03:03:33,320 EXPLAINING A LITTLE BIT ON HOW 4426 03:03:33,320 --> 03:03:34,640 WE WORK WITH THE FLUID 4427 03:03:34,640 --> 03:03:34,960 BIOMARKERS. 4428 03:03:34,960 --> 03:03:38,080 IT'S BEEN AN EXCITING TIME TO 4429 03:03:38,080 --> 03:03:40,600 WORK IN THIS FIELD. 4430 03:03:40,600 --> 03:03:45,720 ESPECIALLY DURING RECENT YEARS. 4431 03:03:45,720 --> 03:03:50,080 BASICALLY THE SLIDE YOU SEE THE 4432 03:03:50,080 --> 03:03:52,120 CHARACTERISTIC OF THE UNDERLYING 4433 03:03:52,120 --> 03:03:58,000 CAUSE RELEASES FILAMENT PROTEINS 4434 03:03:58,000 --> 03:04:02,960 HIGHLY COMMON IN ALS AND 4435 03:04:02,960 --> 03:04:13,520 THROUGHOUT THE YEARS THE BASIC 4436 03:04:15,600 --> 03:04:16,720 IDEA FROM THE BIOMARKERS HAVE 4437 03:04:16,720 --> 03:04:19,040 BEEN ALLUDED TO IN SEVERAL TALKS 4438 03:04:19,040 --> 03:04:23,000 AND YOU SEE THE TIP OF THE 4439 03:04:23,000 --> 03:04:28,120 ICEBERG BUT THE SYMPTOMS ARE 4440 03:04:28,120 --> 03:04:31,840 CAUSED BY FACTORS THAT RESULT IN 4441 03:04:31,840 --> 03:04:36,320 BIO CHEMICAL CHANGES. 4442 03:04:36,320 --> 03:04:41,640 SO AND YOU SEE A NUMBER OF THESE 4443 03:04:41,640 --> 03:04:42,280 BIOMARKERS THAT HAVE BEEN 4444 03:04:42,280 --> 03:04:46,160 DEVELOPED IN RELATION TO ALS. 4445 03:04:46,160 --> 03:04:50,840 YOU SEE SERUM LEVELS THAT START 4446 03:04:50,840 --> 03:04:52,000 TO INCREASE YEARS BEFORE 4447 03:04:52,000 --> 03:04:56,320 CLINICAL ONSET OF ALS AND SEE 4448 03:04:56,320 --> 03:04:56,880 THE LEVELS ARE RELATIVELY 4449 03:04:56,880 --> 03:05:07,040 STABLE. 4450 03:05:08,640 --> 03:05:12,880 AND YOU CAN SEE THE MUTATIONS 4451 03:05:12,880 --> 03:05:14,200 AND THEN IN THE LOWER RIGHT YOU 4452 03:05:14,200 --> 03:05:23,000 SEE VERY EXCITING MARKER WITH A 4453 03:05:23,000 --> 03:05:24,040 MEASUREMENT IN URINE. 4454 03:05:24,040 --> 03:05:26,040 IN ALS WE HAVE MULTIPLE 4455 03:05:26,040 --> 03:05:27,840 BIOMARKERS AT HAND BUT NEED TO 4456 03:05:27,840 --> 03:05:29,480 DEVELOP NEW BIOMARKERS AND SINCE 4457 03:05:29,480 --> 03:05:31,800 THERE'S BEEN SUBFORMS OF ALS WE 4458 03:05:31,800 --> 03:05:37,080 NEED TO DISCOVER BIOMARKERS TO 4459 03:05:37,080 --> 03:05:41,760 REFLECT THE SPECIFIC AND THIS IS 4460 03:05:41,760 --> 03:05:43,320 MY FAVORITE SLIDE AND I OFTEN 4461 03:05:43,320 --> 03:05:47,480 RETURN TO THIS WHEN I THINK 4462 03:05:47,480 --> 03:05:48,160 ABOUT NEURODEGENERATIVE DISEASE 4463 03:05:48,160 --> 03:05:50,160 AND THIS IS AN EXAMPLE OF A 4464 03:05:50,160 --> 03:05:54,960 SUCCESSFUL TREATMENT OF A 4465 03:05:54,960 --> 03:05:57,640 NEURODEGENERATIVE AND SPINAL 4466 03:05:57,640 --> 03:06:00,680 MUSCULAR ATROPHY AND YOU HAVE A 4467 03:06:00,680 --> 03:06:02,520 PROTEIN THAT AFFECTS THE 4468 03:06:02,520 --> 03:06:06,000 SURVIVAL OF MOTOR NEURONS AND 4469 03:06:06,000 --> 03:06:12,080 YOU CAN STABILIZE THE PROTEIN BY 4470 03:06:12,080 --> 03:06:14,400 INJECTING OR GIVING A NUCLEOTIDE 4471 03:06:14,400 --> 03:06:15,240 YOU CAN RESTORE EXPRESSION OF 4472 03:06:15,240 --> 03:06:17,840 THE PROTEIN AND THE KIDS YOU SEE 4473 03:06:17,840 --> 03:06:23,880 HOW YOUNG THEY ARE IN THIS CASE, 4474 03:06:23,880 --> 03:06:30,600 THE KIDS YOU CAN SE THE LEVELS 4475 03:06:30,600 --> 03:06:31,440 DROP RAPIDLY. 4476 03:06:31,440 --> 03:06:34,600 AND OF COURSE THIS IS WHAT WE'RE 4477 03:06:34,600 --> 03:06:45,000 KNOWN FOR SEEING IN ALS. 4478 03:06:59,360 --> 03:07:02,200 AND WE'RE STARTING TO SEE THIS 4479 03:07:02,200 --> 03:07:05,480 APPEAR IN THE ALZHEIMER'S 4480 03:07:05,480 --> 03:07:09,840 DISEASE AND YOU SEE 4481 03:07:09,840 --> 03:07:14,000 DOSE-DEPENDENT LURING OF 4482 03:07:14,000 --> 03:07:14,520 FILAM 4483 03:07:14,520 --> 03:07:14,800 FILAMENT. 4484 03:07:14,800 --> 03:07:19,080 IT STARTS TO LOOK PROMISING. 4485 03:07:19,080 --> 03:07:20,920 THIS IS WHY THIS IS A HIGH 4486 03:07:20,920 --> 03:07:26,120 PRIORITY. 4487 03:07:26,120 --> 03:07:31,680 THEN I I'LL HAND IT OVER TO 4488 03:07:31,680 --> 03:07:31,880 STEVE. 4489 03:07:31,880 --> 03:07:33,600 >>THANK YOU. 4490 03:07:33,600 --> 03:07:36,680 I'LL GIVE A QUICK OVERVIEW OF A 4491 03:07:36,680 --> 03:07:39,640 RICH SUBJECT, HOW ONE WOULD FIND 4492 03:07:39,640 --> 03:07:41,960 THE MOLECULAR INVENTION OF 4493 03:07:41,960 --> 03:07:45,240 DISEASE ONCE WE HAVE THE 4494 03:07:45,240 --> 03:07:47,080 RESOURCES IN THE INDUSTRY AND 4495 03:07:47,080 --> 03:07:48,320 THE WORLD. 4496 03:07:48,320 --> 03:07:51,080 ULTIMATELY YOU WANT TO MIMIC THE 4497 03:07:51,080 --> 03:07:52,160 DISEASE SOMEHOW. 4498 03:07:52,160 --> 03:07:55,040 SOME MAY DO DISEASE IN A DISH 4499 03:07:55,040 --> 03:07:56,720 MODELS TAKING CELLS FROM AN 4500 03:07:56,720 --> 03:08:00,720 ANIMAL MODEL OF DISEASE OR CELLS 4501 03:08:00,720 --> 03:08:02,400 DIRECTLY FROM A HUMAN PATIENT 4502 03:08:02,400 --> 03:08:04,680 AND TRY TO PERTURB THE GENES AND 4503 03:08:04,680 --> 03:08:06,600 THE CHALLENGE IS HOW DOES IT 4504 03:08:06,600 --> 03:08:08,240 RELATE TO HUMAN DISEASE? 4505 03:08:08,240 --> 03:08:13,520 SIMILARLY, ANIMAL MODELS OF 4506 03:08:13,520 --> 03:08:19,560 DISEASE WE CAN PUT HUMAN-DISEASE 4507 03:08:19,560 --> 03:08:23,040 CAUSES CELLS INTO MICE AND FLIES 4508 03:08:23,040 --> 03:08:25,200 AND MANIPULATE THESE 4509 03:08:25,200 --> 03:08:27,640 PHARMACOLOGICALLY OR WITH TEST 4510 03:08:27,640 --> 03:08:27,960 THERAPEUTICS. 4511 03:08:27,960 --> 03:08:30,800 KEY QUESTION, HOW'S IT RELATE TO 4512 03:08:30,800 --> 03:08:31,280 HUMAN DISEASE? 4513 03:08:31,280 --> 03:08:33,560 WHAT'S BEEN VERY FRUITFUL IS 4514 03:08:33,560 --> 03:08:36,720 LOOKING AT HUMAN GENETICS AND 4515 03:08:36,720 --> 03:08:40,160 GENOMICS AMONG HEREDITARY CASES 4516 03:08:40,160 --> 03:08:46,720 AS YOU SEE WITH CHROMOSOME 9 AND 4517 03:08:46,720 --> 03:08:49,200 CAN YOU ZERO INTO THE MOLECULAR 4518 03:08:49,200 --> 03:08:50,040 CAUSE AND THEN HAVE YOU 4519 03:08:50,040 --> 03:08:52,160 SOMETHING TO WORK ON AND THERE'S 4520 03:08:52,160 --> 03:08:55,840 A CAVEAT YOU CANNOT DO ANYTHING 4521 03:08:55,840 --> 03:08:56,680 OBSERVATIONAL AND CAN'T 4522 03:08:56,680 --> 03:09:01,360 MANIPULATE OR INTERVENE. 4523 03:09:01,360 --> 03:09:05,000 ARE ONES YOU HAVE IDEAS FOR A 4524 03:09:05,000 --> 03:09:11,280 THERAPEUTIC HYPOTHESIS IT'S 4525 03:09:11,280 --> 03:09:15,720 BEWILDERING AND ASPIRIN PEPTIDES 4526 03:09:15,720 --> 03:09:20,240 AND EVER INCREASINGLY NUCLEIC 4527 03:09:20,240 --> 03:09:27,320 ASSETS AND WHEN SHOWN IS A SHORT 4528 03:09:27,320 --> 03:09:30,440 INTERFERING RNA OLIGOSACCHARIDES 4529 03:09:30,440 --> 03:09:34,160 AND GENE EDITING AND GENE 4530 03:09:34,160 --> 03:09:35,680 THERAPIES WHERE ONE WOULD 4531 03:09:35,680 --> 03:09:37,040 MANUFACTURE CELLS TO REGROW 4532 03:09:37,040 --> 03:09:37,280 TISSUE. 4533 03:09:37,280 --> 03:09:39,680 ONE HAS PLUSES AND MINUS. 4534 03:09:39,680 --> 03:09:41,360 THE IDEAL DRUG WILL BE ABLE TO 4535 03:09:41,360 --> 03:09:45,800 BE TAKEN INFREQUENTLY, TAKEN IN 4536 03:09:45,800 --> 03:09:49,160 A PILL, DIFFUSED THROUGHOUT THE 4537 03:09:49,160 --> 03:09:52,840 BRAIN AND HAVE SPECIFICITY TO 4538 03:09:52,840 --> 03:09:58,160 THE TARGET AND MANUFACTURING. 4539 03:09:58,160 --> 03:10:00,280 EACH HAVE PLUSES AND MINUSES AND 4540 03:10:00,280 --> 03:10:02,360 IT'S A WONDERFUL MESH THAT'S 4541 03:10:02,360 --> 03:10:07,360 NEEDED BETWEEN TARGET HYPOTHESES 4542 03:10:07,360 --> 03:10:08,600 AND INDUSTRY WHO WILL TAKE WHAT 4543 03:10:08,600 --> 03:10:11,360 IS THE BEST ONE AND TRY TO MATCH 4544 03:10:11,360 --> 03:10:13,440 IT WITH THE APPROPRIATE 4545 03:10:13,440 --> 03:10:13,760 ENGINEERING. 4546 03:10:13,760 --> 03:10:15,440 THESE THEN WILL BUILD IN TO 4547 03:10:15,440 --> 03:10:19,600 WHERE WE HAVE EXACTLY WHAT 4548 03:10:19,600 --> 03:10:22,160 ALREADY EXISTS INTO WHAT WE'RE 4549 03:10:22,160 --> 03:10:25,560 PROPOSING AND ONCE YOU HAVE A 4550 03:10:25,560 --> 03:10:27,000 TARGET AND IT WILL ENABLE 4551 03:10:27,000 --> 03:10:27,280 BIOMARKERS. 4552 03:10:27,280 --> 03:10:28,680 THIS WILL ENABLE US TO SELECT 4553 03:10:28,680 --> 03:10:32,680 THE PATIENTS AND MONITOR PATIENT 4554 03:10:32,680 --> 03:10:37,880 PROGRESSION PERHAPS EVEN SELECT 4555 03:10:37,880 --> 03:10:44,200 PATIENTS PRESYMPTOMATICALLY AND 4556 03:10:44,200 --> 03:10:45,040 APPRECIATION ORGANIZATIONS, 4557 03:10:45,040 --> 03:10:49,560 INDUSTRY AND OF COURSE THE FDA 4558 03:10:49,560 --> 03:10:50,880 AND APPROPRIATE REGULATORY 4559 03:10:50,880 --> 03:10:52,200 COMMANDS. 4560 03:10:52,200 --> 03:11:00,320 I'LL PASS IT ON. 4561 03:11:00,320 --> 03:11:06,400 >>I'M JIM WILSON AND HAVE BEEN 4562 03:11:06,400 --> 03:11:08,680 ASKED TO COMMENT AND WE HEARD 4563 03:11:08,680 --> 03:11:14,880 IMPORTANT PRINCIPLES 4564 03:11:14,880 --> 03:11:20,720 HISTORICALLY ABOUT HOW ABOUT THE 4565 03:11:20,720 --> 03:11:31,200 TRANSLATION AND AN CLINICAL 4566 03:11:33,040 --> 03:11:37,480 RESEARCH AND WE HEARDS THE 4567 03:11:37,480 --> 03:11:40,880 DEPRESSING OR REALISTIC VIEW OF 4568 03:11:40,880 --> 03:11:41,640 HOW HOW LONG IT TAKES AND HOW 4569 03:11:41,640 --> 03:11:48,600 MUCH IT COSTS AND WE'RE IN AN 4570 03:11:48,600 --> 03:11:52,160 ERA NOW ABOUT A KNEW MODALITY OF 4571 03:11:52,160 --> 03:12:02,680 GENETIC MEDICINES AND YOU CAN 4572 03:12:08,760 --> 03:12:11,120 LEVERAGE FACTORS AND IT TURNS 4573 03:12:11,120 --> 03:12:15,680 OUT THE ACADEMY IS POSITIONED 4574 03:12:15,680 --> 03:12:21,680 THAN BEFORE TO TAKE GENETIC 4575 03:12:21,680 --> 03:12:27,920 MEDICINES AND TAKE GENETIC 4576 03:12:27,920 --> 03:12:34,040 MEDICINES INTO THE CLINIC. 4577 03:12:34,040 --> 03:12:35,800 AND WHAT THIS ENABLED US TO DO 4578 03:12:35,800 --> 03:12:42,040 NOT ONLY US BUT THE WHOLE ECO 4579 03:12:42,040 --> 03:12:48,680 SYSTEM IS THAT WE'VE BEEN ABLE 4580 03:12:48,680 --> 03:12:54,480 TO DERISK THE STRATEGIES MAKING 4581 03:12:54,480 --> 03:12:56,640 IT MORE ATTRACTIVE. 4582 03:12:56,640 --> 03:12:57,920 AND WE AGGRESSIVELY MORE THAN 4583 03:12:57,920 --> 03:13:00,200 ANYONE FIND WAYS TO TARGET WITH 4584 03:13:00,200 --> 03:13:02,560 INDUSTRY IN OUR TRANSLATIONAL 4585 03:13:02,560 --> 03:13:07,080 RESEARCH BUT THERE ARE TIMES I 4586 03:13:07,080 --> 03:13:09,360 WOULD ARGUE THE COMMUNITY MAY 4587 03:13:09,360 --> 03:13:11,800 NEED TO MOVE THE BAR FURTHER 4588 03:13:11,800 --> 03:13:16,560 INTO FIRST IN HUMAN STUDIES BY 4589 03:13:16,560 --> 03:13:19,000 OURSELVES WITH INFRASTRUCTURE 4590 03:13:19,000 --> 03:13:21,640 THAT HAS BEEN DESCRIBED OR THAT 4591 03:13:21,640 --> 03:13:23,640 EXISTS SO THAT WE CAN THEN 4592 03:13:23,640 --> 03:13:29,080 DETERMINE WHETHER THE CONCEPTS 4593 03:13:29,080 --> 03:13:39,240 ARE VALID OR NOT IT'S BEEN OUR 4594 03:13:39,240 --> 03:13:41,320 EXPERIENCE AT PENN WHEN WE DO 4595 03:13:41,320 --> 03:13:49,280 THAT WE GET INVESTING FROM. 4596 03:13:49,280 --> 03:13:52,080 NADIA. 4597 03:13:52,080 --> 03:13:54,080 >>I'M HERE TO PROVIDE THE 4598 03:13:54,080 --> 03:13:55,280 COMMUNITY PERSPECTIVE ON WHAT IS 4599 03:13:55,280 --> 03:13:55,520 PROPOSED. 4600 03:13:55,520 --> 03:13:57,440 WE DON'T HAVE TIME ON OUR SIDE 4601 03:13:57,440 --> 03:13:58,160 IN THIS DISEASE. 4602 03:13:58,160 --> 03:14:00,280 THERE'S A MEDIAN SURVIVAL OF 30 4603 03:14:00,280 --> 03:14:01,880 MONTHS FROM FIRST SYMPTOM. 4604 03:14:01,880 --> 03:14:06,960 THERE'S A LOT OF URGENCY. 4605 03:14:06,960 --> 03:14:12,400 AND SO MANY OTHERS EMPHASIZED 4606 03:14:12,400 --> 03:14:15,120 THERE'S A 30% GENETIC COMPONENT 4607 03:14:15,120 --> 03:14:16,480 AND WE'RE ONLY ON THE VERGE OF 4608 03:14:16,480 --> 03:14:18,520 TREATMENT AND WE HAVE SOME IN 4609 03:14:18,520 --> 03:14:22,280 THE PIPELINE BUT NOTHING 4610 03:14:22,280 --> 03:14:23,200 DEFINITE. 4611 03:14:23,200 --> 03:14:28,680 FOR EVERYONE ELSE THAT HAS A 4612 03:14:28,680 --> 03:14:30,520 SPORADIC FORM OF THE DISEASE WE 4613 03:14:30,520 --> 03:14:32,000 NEED A BETTER APPROACH TO ALS. 4614 03:14:32,000 --> 03:14:33,520 IT'S DIFFERENT IN HOW IT STARTS 4615 03:14:33,520 --> 03:14:35,520 AND PROGRESSES AND UNDERLYING 4616 03:14:35,520 --> 03:14:38,720 CAUSES FROM PERSON TO PERSON. 4617 03:14:38,720 --> 03:14:40,920 THAT'S THE HETEROGENEITY PROBLEM 4618 03:14:40,920 --> 03:14:43,800 IN ALS. 4619 03:14:43,800 --> 03:14:46,000 WE HAVE ABOUT 100 INTERVENTIONAL 4620 03:14:46,000 --> 03:14:47,320 TRIALS AND THERE'S PROMISE IF 4621 03:14:47,320 --> 03:14:49,120 YOU TALK TO ANYBODY THEY'LL TELL 4622 03:14:49,120 --> 03:14:50,600 YOU THAT'S MORE CLINICAL TRIALS 4623 03:14:50,600 --> 03:14:52,160 THAN WE'VE HAD INVEST PAST AND 4624 03:14:52,160 --> 03:14:56,720 HAVE BETTER SCIENCE IN THE 4625 03:14:56,720 --> 03:15:02,080 TRIALS AND THOSE CLOSEST TO 4626 03:15:02,080 --> 03:15:04,360 BEING ACCESSIBLE TO COMMUNITY 4627 03:15:04,360 --> 03:15:06,000 AND TO HAVE SCIENCE EXCEL BEHIND 4628 03:15:06,000 --> 03:15:07,160 IT THAT'S WHERE THE 4629 03:15:07,160 --> 03:15:08,720 TRANSLATIONAL RESEARCH COMES IN 4630 03:15:08,720 --> 03:15:09,920 TO MAKE A DIFFERENCE. 4631 03:15:09,920 --> 03:15:12,160 THE PURPOSE OF THIS WORKING 4632 03:15:12,160 --> 03:15:14,560 GROUP WAS TO PROPOSE WAYS TO 4633 03:15:14,560 --> 03:15:15,760 TRANSLATE EARLY RESEARCH 4634 03:15:15,760 --> 03:15:17,400 FINDINGS INTO BETTER TRIALS AND 4635 03:15:17,400 --> 03:15:17,760 TREATMENTS. 4636 03:15:17,760 --> 03:15:20,080 HOW DO WE PUSH THE ALS TREATMENT 4637 03:15:20,080 --> 03:15:21,000 PIPELINE FOR EVERYONE LEAVING 4638 03:15:21,000 --> 03:15:21,680 WITH ALS. 4639 03:15:21,680 --> 03:15:28,680 HOW DO WE OVERCOME HETEROGENEITY 4640 03:15:28,680 --> 03:15:32,680 AND THE VARIABILITY IN THE 4641 03:15:32,680 --> 03:15:34,840 PROGRESSION AND ONSET. 4642 03:15:34,840 --> 03:15:36,760 THE PURPOSE OF THE GROUP IS TO 4643 03:15:36,760 --> 03:15:39,080 OUTLINE MORE TARGETED TREATMENTS 4644 03:15:39,080 --> 03:15:41,240 FOR ALL TYPES OF ALS. 4645 03:15:41,240 --> 03:15:42,200 TRANSLATIONAL RESEARCH IS THE 4646 03:15:42,200 --> 03:15:43,920 BRIDGE BETWEEN EARLY SCIENCE AND 4647 03:15:43,920 --> 03:15:44,240 TREATMENTS. 4648 03:15:44,240 --> 03:15:46,360 SO HOW CAN WE USE TRANSLATIONAL 4649 03:15:46,360 --> 03:15:49,160 RESEARCH TO OVERCOME THE 4650 03:15:49,160 --> 03:15:51,760 HETEROGENEITY OF ALS. 4651 03:15:51,760 --> 03:15:55,040 CONCERTED DATA AND BIO SAMPLE 4652 03:15:55,040 --> 03:15:55,800 COLLECTION FROM PEOPLE LIVING 4653 03:15:55,800 --> 03:15:57,080 WITH ALS THE MORE THE BETTER AND 4654 03:15:57,080 --> 03:15:59,000 SHARING THE INFORMATION AND 4655 03:15:59,000 --> 03:16:01,800 SAMPLES OPENLY AND BROADLY WITH 4656 03:16:01,800 --> 03:16:03,200 THE RESEARCH COMMUNITY AND 4657 03:16:03,200 --> 03:16:06,240 INDUSTRY TO FIND MORE BIOMARKERS 4658 03:16:06,240 --> 03:16:08,040 AND SOMETHING MEASURABLE TO TELL 4659 03:16:08,040 --> 03:16:09,920 US WHAT'S HAPPENING WITH AN 4660 03:16:09,920 --> 03:16:11,200 INDIVIDUAL AND WHEN AND THE 4661 03:16:11,200 --> 03:16:13,080 OUTLINES BY THE WORKING GROUP 4662 03:16:13,080 --> 03:16:15,320 INCLUDED CREATING A NETWORK OF 4663 03:16:15,320 --> 03:16:19,800 ALS CENTERS OF EXCELLENCE, BIO 4664 03:16:19,800 --> 03:16:22,000 SAMPLE AND FOR NIH TO SUPPORT 4665 03:16:22,000 --> 03:16:22,840 THERAPEUTICS FROM THE CLEAR 4666 03:16:22,840 --> 03:16:24,360 STRATEGY AND PROCESS. 4667 03:16:24,360 --> 03:16:26,520 THIS WILL BE THE MOST EFFICIENT 4668 03:16:26,520 --> 03:16:28,080 WAY TO UNDERSTAND ALS BETTER 4669 03:16:28,080 --> 03:16:30,320 WHICH WILL THEN LEAD TO BETTER 4670 03:16:30,320 --> 03:16:30,920 TREATMENTS WITH STRONGER 4671 03:16:30,920 --> 03:16:33,560 RATIONALE AND GUIDE US TO THE 4672 03:16:33,560 --> 03:16:35,200 PATH OF MATCHING THE RIGHT 4673 03:16:35,200 --> 03:16:36,680 TREATMENT TO A PARTICULAR PERSON 4674 03:16:36,680 --> 03:16:40,680 WITH ALS NOT JUST BASED ON 4675 03:16:40,680 --> 03:16:42,240 GENETIC SUBTYPES. 4676 03:16:42,240 --> 03:16:46,160 IT'S ALSO THE PATH TO FASTER 4677 03:16:46,160 --> 03:16:48,720 DIAGNOSIS AND MEASURING 4678 03:16:48,720 --> 03:16:55,160 PROGRESSION AND THE EXPERIMENTS 4679 03:16:55,160 --> 03:16:56,520 WILL AMEND OUR EXPERIMENTAL 4680 03:16:56,520 --> 03:16:57,560 PIPELINE WITH THE ALS COMMUNITY. 4681 03:16:57,560 --> 03:17:06,760 THANK YOU, EVERYBODY. 4682 03:17:06,760 --> 03:17:07,280 >>THANK YOU. 4683 03:17:07,280 --> 03:17:08,760 WE'RE NOW GOING TO TURN IT OVER 4684 03:17:08,760 --> 03:17:12,680 TO QUESTIONS AND POST ANY 4685 03:17:12,680 --> 03:17:16,440 QUESTIONS HAVE YOU IN THE Q&A 4686 03:17:16,440 --> 03:17:18,480 AREA OF ZOOM AND WE'LL GET TO AS 4687 03:17:18,480 --> 03:17:21,080 MANY AS WE CAN NOW. 4688 03:17:21,080 --> 03:17:22,920 I'LL START WITH A QUESTION THAT 4689 03:17:22,920 --> 03:17:26,040 CAME IN AHEAD OF TIME WHICH I'LL 4690 03:17:26,040 --> 03:17:27,320 DIRECT TO NEIL. 4691 03:17:27,320 --> 03:17:31,160 THE QUESTION READS, WHY AREN'T 4692 03:17:31,160 --> 03:17:36,960 DRUGS BEING TESTED AND SHOULDN'T 4693 03:17:36,960 --> 03:17:44,680 WE TREAT AT EARLIER PROGRESSION 4694 03:17:44,680 --> 03:17:47,160 PLEASE LOOK TOWARDS THOSE 4695 03:17:47,160 --> 03:17:47,760 WITHOUT DISEASE. 4696 03:17:47,760 --> 03:17:52,440 >>THANK YOU. 4697 03:17:52,440 --> 03:17:57,520 THIS IS A GREAT QUESTION 4698 03:17:57,520 --> 03:17:59,360 IMPORTANT QUESTION AND ONE THAT 4699 03:17:59,360 --> 03:18:03,280 IS ING AND MY ANSWER WILL 4700 03:18:03,280 --> 03:18:09,280 PROVIDE HOPE TO THOSE WHO ARE IN 4701 03:18:09,280 --> 03:18:16,040 THIS POSITION. 4702 03:18:16,040 --> 03:18:17,960 THERE'S NO QUESTION TREATING AS 4703 03:18:17,960 --> 03:18:19,680 EARLY IN THE PROCESS WILL LEAD 4704 03:18:19,680 --> 03:18:22,160 TO THE MOST POSITIVE THERAPEUTIC 4705 03:18:22,160 --> 03:18:22,640 OUTCOMES. 4706 03:18:22,640 --> 03:18:24,320 IDEALLY WE WANT TO TREAT 4707 03:18:24,320 --> 03:18:25,280 PATIENTS BEFORE THEY DEVELOP 4708 03:18:25,280 --> 03:18:26,520 SIGNS AND SYMPTOMS OF DISEASE. 4709 03:18:26,520 --> 03:18:29,120 WE DON'T HAVE THE BIOMARKERS NOW 4710 03:18:29,120 --> 03:18:31,120 TO DO THIS IN THE STRATA 4711 03:18:31,120 --> 03:18:34,960 POPULATION BUT WE CAN IN RARE 4712 03:18:34,960 --> 03:18:36,000 GENETIC FORMS OF THE DISEASE. 4713 03:18:36,000 --> 03:18:38,640 THE FIRST STEP IN TREATING THE 4714 03:18:38,640 --> 03:18:42,120 PATIENTS IS IDENTIFYING THEM AND 4715 03:18:42,120 --> 03:18:45,640 FOLLOWING THEM IN THAT 4716 03:18:45,640 --> 03:18:46,720 PRE-SYMPTOMATIC PHASE TO 4717 03:18:46,720 --> 03:18:52,160 IDENTIFY MARKERS THAT ARE 4718 03:18:52,160 --> 03:18:56,280 HARBINGERS OF DISEASE ONSET AND 4719 03:18:56,280 --> 03:18:58,200 FIGURING OUT WHEN TO INTERVENE 4720 03:18:58,200 --> 03:19:06,200 TO PREVENT OR DELAY OR PREVENT 4721 03:19:06,200 --> 03:19:07,480 DISEASE ONSET. 4722 03:19:07,480 --> 03:19:09,320 THIS IS HAPPENING AND TOOK PLACE 4723 03:19:09,320 --> 03:19:11,760 IN THE DISCUSSION THIS MORNING. 4724 03:19:11,760 --> 03:19:13,760 THERE'S EFFORTS IN THE UNITED 4725 03:19:13,760 --> 03:19:17,480 STATES TO IDENTIFY AND 4726 03:19:17,480 --> 03:19:23,440 CHARACTERIZE PRESYMPTOMATIC 4727 03:19:23,440 --> 03:19:25,520 CAREERS ARE OF THE MUTATIONS AND 4728 03:19:25,520 --> 03:19:35,760 ONE WAS LED BY MICHAEL BENNATAR 4729 03:19:35,760 --> 03:19:37,640 EFFORTS AND IT'S ACTIVE IN 4730 03:19:37,640 --> 03:19:37,920 COLUMBIA. 4731 03:19:37,920 --> 03:19:39,760 WE RUN A PROGRAM CALLED THE ALS 4732 03:19:39,760 --> 03:19:42,200 FAMILIES PROJECT AND WE'RE 4733 03:19:42,200 --> 03:19:46,200 WORKING NOW CLOSELY TO PERFORM 4734 03:19:46,200 --> 03:19:49,000 THE ENTITY INITIATIVE CALLED 4735 03:19:49,000 --> 03:19:54,640 PREVENT ALS TO COMBINE AND 4736 03:19:54,640 --> 03:19:56,240 HARMONIZE OUR EFFORTS AND OUR 4737 03:19:56,240 --> 03:19:59,840 EFFORTS TO MORE CENTERS TO 4738 03:19:59,840 --> 03:20:03,280 INVOLVE MORE PATIENTS OF DIVERSE 4739 03:20:03,280 --> 03:20:04,160 BACKGROUNDS, GENETIC AND 4740 03:20:04,160 --> 03:20:13,160 OTHERWISE. 4741 03:20:13,160 --> 03:20:19,800 WE'RE FOLLOWING THE PATIENTS TO 4742 03:20:19,800 --> 03:20:22,880 COLLECT SAMPLES AND DO BIOMARKER 4743 03:20:22,880 --> 03:20:25,200 STUDIES AND IDENTIFY INDIVIDUALS 4744 03:20:25,200 --> 03:20:27,200 WHO WOULD BENEFIT FROM AN EARLY 4745 03:20:27,200 --> 03:20:28,160 INTERVENTION. 4746 03:20:28,160 --> 03:20:38,600 THIS IS HAPPENING ALREADY. 4747 03:20:42,120 --> 03:20:52,160 THERE'S A TRIAL AND THE ATLAS 4748 03:20:52,160 --> 03:20:56,320 TRIAL STARTED ON THE 4749 03:20:56,320 --> 03:20:57,840 LONGITUDINAL STUDY OF 4750 03:20:57,840 --> 03:20:59,640 PRESYMPTOMATIC CARRIES OF THE 4751 03:20:59,640 --> 03:21:01,640 MUTATION AND BASED ON THE BASIS 4752 03:21:01,640 --> 03:21:04,200 OF THAT INFORMATION OF THE TRIAL 4753 03:21:04,200 --> 03:21:14,320 DESIGN WAS BIASED. 4754 03:21:14,320 --> 03:21:20,360 IN MY OWN TRIALS WE'RE WORKING 4755 03:21:20,360 --> 03:21:23,840 ON A TRIAL AND WE BEGAN TO 4756 03:21:23,840 --> 03:21:27,120 EXPAND ACCESS TO THE PROGRAM AND 4757 03:21:27,120 --> 03:21:32,600 AS MENTIONED BY OUR GROUP 4758 03:21:32,600 --> 03:21:36,360 EARLIER WE WERE ABLE TO SPONSOR 4759 03:21:36,360 --> 03:21:36,920 THE TRIAL. 4760 03:21:36,920 --> 03:21:39,680 BUT IN OUR EXPANDED ACCESS STUDY 4761 03:21:39,680 --> 03:21:44,440 WE TREATED FIRST SYMPTOMATIC 4762 03:21:44,440 --> 03:21:46,200 INDIVIDUALS BUT MOVED 4763 03:21:46,200 --> 03:21:50,120 PROGRESSIVELY BACK TO EARLIER 4764 03:21:50,120 --> 03:21:58,160 STAGE PEOPLE AND THE 4765 03:21:58,160 --> 03:21:58,920 PRESYMPTOMATIC INDIVIDUAL OF THE 4766 03:21:58,920 --> 03:21:59,160 DISEASE. 4767 03:21:59,160 --> 03:22:03,560 THIS NEEDS TO HAPPEN AT GREATER 4768 03:22:03,560 --> 03:22:05,600 SCALE AND WITH THE FOUNDATION WE 4769 03:22:05,600 --> 03:22:07,920 AT COLUMBIA LAUNCHED AN EFFORT 4770 03:22:07,920 --> 03:22:10,200 CALLED SILENCE ALS TAKING ON THE 4771 03:22:10,200 --> 03:22:17,280 INDIVIDUAL RARE CASES DEVELOPING 4772 03:22:17,280 --> 03:22:22,360 THERAPEUTICS EVEN AS SMALL AS A 4773 03:22:22,360 --> 03:22:28,160 CASE WITH A THERAPEUTIC THAT 4774 03:22:28,160 --> 03:22:32,680 SEEMS TO WORK IN SYMPTOMATIC 4775 03:22:32,680 --> 03:22:35,480 CHALLENGE IS TO GO BACK TO 4776 03:22:35,480 --> 03:22:40,200 COHORT ARE AT RISK WITH 4777 03:22:40,200 --> 03:22:50,600 PRESYMPTOMATIC SYMPTOMS. 4778 03:22:51,760 --> 03:22:53,600 THE NEXT QUESTION, STACIE, I'LL 4779 03:22:53,600 --> 03:22:56,240 DIRECT TO YOU, WHY DO YOU SAY 4780 03:22:56,240 --> 03:22:57,520 YOU ARE RESEARCHING THERAPIES 4781 03:22:57,520 --> 03:23:00,680 AND NOT POTENTIAL CURE LIKE IN 4782 03:23:00,680 --> 03:23:01,000 CANCER? 4783 03:23:01,000 --> 03:23:01,760 SHOULDN'T THE RESEARCH FOCUS BE 4784 03:23:01,760 --> 03:23:07,920 ON FINDING THE CURE? 4785 03:23:07,920 --> 03:23:16,560 >>ULTIMATELY ABSOLUTELY BUT I 4786 03:23:16,560 --> 03:23:24,920 THINK THAT WE ARE AWARE THEY 4787 03:23:24,920 --> 03:23:29,040 MOVE FORWARD IN A STEPWISE 4788 03:23:29,040 --> 03:23:29,520 FASHION. 4789 03:23:29,520 --> 03:23:32,680 CANCER HAS BEEN EXTENDED BY A 4790 03:23:32,680 --> 03:23:34,680 QUALITY OF LIFE IT'S ONLY A 4791 03:23:34,680 --> 03:23:37,360 SMALL NUMBER OF CANCERS THAT WE 4792 03:23:37,360 --> 03:23:37,560 CURE. 4793 03:23:37,560 --> 03:23:40,200 AND THERE'S SIGNIFICANT BENEFIT 4794 03:23:40,200 --> 03:23:50,440 TO INCREASING QUALITY OF LIFE, 4795 03:23:50,440 --> 03:23:52,280 INCREASING LIFE SPAN AND QUALITY 4796 03:23:52,280 --> 03:23:53,480 OF LIFE. 4797 03:23:53,480 --> 03:23:55,640 ULTIMATELY PREVENT HOW I THINK 4798 03:23:55,640 --> 03:23:58,000 WE'LL GET TO THE CURE TO TREAT 4799 03:23:58,000 --> 03:23:58,200 EARLY. 4800 03:23:58,200 --> 03:24:02,320 BUT THE TIME SOMEONE IS SHOWING 4801 03:24:02,320 --> 03:24:02,960 SYMPTOMS, THERE'S ALREADY ALL 4802 03:24:02,960 --> 03:24:03,720 RIGHT DAMAGE THAT'S BEEN DONE IN 4803 03:24:03,720 --> 03:24:04,160 THE BRAIN. 4804 03:24:04,160 --> 03:24:09,400 I THINK THE FIRST STEP OF WHAT 4805 03:24:09,400 --> 03:24:16,680 WE'RE TRYING TO DO IS STOP THE 4806 03:24:16,680 --> 03:24:17,560 PROGRESSION OF EARLY STAGE 4807 03:24:17,560 --> 03:24:20,480 DISEASE THAT WOULD BE INCREDIBLY 4808 03:24:20,480 --> 03:24:20,800 SIGNIFICANT. 4809 03:24:20,800 --> 03:24:22,560 I THINK THAT'S WHAT WE'RE 4810 03:24:22,560 --> 03:24:23,480 STRIVING FOR. 4811 03:24:23,480 --> 03:24:26,560 YOU'LL HAVE TO GET IT RIGHT AND 4812 03:24:26,560 --> 03:24:28,320 WE HAVE TO LEARN A LOT HERE. 4813 03:24:28,320 --> 03:24:33,800 SO I THINK EACH STEP WE MAKE 4814 03:24:33,800 --> 03:24:35,760 EVEN IF PERHAPS OUR FIRST 4815 03:24:35,760 --> 03:24:38,560 THERAPEUTICS ARE SLOWING THE 4816 03:24:38,560 --> 03:24:40,760 PROGRESSION OR STOPPING THE 4817 03:24:40,760 --> 03:24:42,280 PROGRESSION AND WALK IT BACK 4818 03:24:42,280 --> 03:24:45,440 WE'LL HAVE A MORE SIGNIFICANT. 4819 03:24:45,440 --> 03:24:48,680 THERE'S ABSOLUTELY A GOAL 4820 03:24:48,680 --> 03:24:49,720 EVENTUALLY THAT WE WILL HAVE A 4821 03:24:49,720 --> 03:24:58,960 QUOTE, CURE. 4822 03:24:58,960 --> 03:25:04,200 ANYTHING TO INCREASE QUALITY OF 4823 03:25:04,200 --> 03:25:10,600 LIFE IN THE INTERIM HELPS. 4824 03:25:10,600 --> 03:25:12,680 I'M GOING READ THE NEXT 4825 03:25:12,680 --> 03:25:15,760 QUESTION. 4826 03:25:15,760 --> 03:25:16,320 THIS CAME UP IN THE EARLIER 4827 03:25:16,320 --> 03:25:19,080 SESSION AS WELL. 4828 03:25:19,080 --> 03:25:22,960 SO THE QUESTION IS READS, I'M A 4829 03:25:22,960 --> 03:25:28,040 BIG BELIEVER IN CRISPR PLATFORM 4830 03:25:28,040 --> 03:25:29,960 FOR RESOLVING ISSUES. 4831 03:25:29,960 --> 03:25:33,440 HAS THERE BEEN PROGRESS IN 4832 03:25:33,440 --> 03:25:35,800 CRISPR TO EXTEND WHAT THE 4833 03:25:35,800 --> 03:25:39,200 MUTATIONS HAVE? 4834 03:25:39,200 --> 03:25:42,680 I'M GOING TO MAYBE STEPHAN, DO 4835 03:25:42,680 --> 03:25:45,720 YOU WANT TO TALK A BIT ABOUT 4836 03:25:45,720 --> 03:25:46,280 THIS? 4837 03:25:46,280 --> 03:25:47,840 YOU'VE BEEN INVOLVED WITH 4838 03:25:47,840 --> 03:25:48,120 COMPANIES. 4839 03:25:48,120 --> 03:25:51,000 >>NO QUESTION, CRISPR IS A VERY 4840 03:25:51,000 --> 03:25:52,800 PROMISING MODALITY ACROSS MANY 4841 03:25:52,800 --> 03:25:53,720 DISEASES. 4842 03:25:53,720 --> 03:26:01,240 AND IT'S STARTING TO SHOW REALAL 4843 03:26:01,240 --> 03:26:01,480 PROMISE. 4844 03:26:01,480 --> 03:26:03,480 IN PRINCIPLE, YES, GOING BACK TO 4845 03:26:03,480 --> 03:26:05,280 MATCH THE MODALITY TO THE 4846 03:26:05,280 --> 03:26:05,520 DISEASE. 4847 03:26:05,520 --> 03:26:06,960 THE BRAIN IS TENDS TO BE ONE OF 4848 03:26:06,960 --> 03:26:09,280 THE LAST THINGS PEOPLE WANT TO 4849 03:26:09,280 --> 03:26:10,600 GET INTO. 4850 03:26:10,600 --> 03:26:12,680 IT'S HARD TO GET DRUGS INTO IT 4851 03:26:12,680 --> 03:26:15,840 AND YOU DON'T KNOW HOW THE BRAIN 4852 03:26:15,840 --> 03:26:20,680 RESPONDS WITH NUCLEIC ACIDS IN 4853 03:26:20,680 --> 03:26:24,680 IT AND PEOPLE ARE LOOKING AT IN. 4854 03:26:24,680 --> 03:26:28,680 IN ADDITION TO CRISPR THERE MAY 4855 03:26:28,680 --> 03:26:31,880 BE SMALL MOLECULE APPROACH AND 4856 03:26:31,880 --> 03:26:34,240 RNA INTERFERENCE APPROACHES AND 4857 03:26:34,240 --> 03:26:36,400 WHAT HAS BEEN IN THE CLINIC ARE 4858 03:26:36,400 --> 03:26:37,400 MANY APPROACH. 4859 03:26:37,400 --> 03:26:38,640 IT'S ON THE WAY. 4860 03:26:38,640 --> 03:26:44,680 IF CRISPR CAN BE ENGINEERED TO 4861 03:26:44,680 --> 03:26:47,600 C972 PEOPLE WILL TRY IT. 4862 03:26:47,600 --> 03:26:49,320 RIGHT NOW CRISPR IS AT A LITTLE 4863 03:26:49,320 --> 03:26:51,280 BIT MORE ACCESSIBLE DISEASES. 4864 03:26:51,280 --> 03:27:01,520 >>THANK YOU. 4865 03:27:10,280 --> 03:27:12,680 STEFAN. 4866 03:27:12,680 --> 03:27:23,240 >>ULTIMATELY THERE'S DELIVERY 4867 03:27:33,400 --> 03:27:38,160 TO THE RIGHT CELL. 4868 03:27:38,160 --> 03:27:40,400 IT TURNS OUT THAT AT LEAST A 4869 03:27:40,400 --> 03:27:43,240 SUBSET OF CELLS THAT MAY BE 4870 03:27:43,240 --> 03:27:45,520 NECESSARY TO TREAT FOR ALS, 4871 03:27:45,520 --> 03:27:48,680 SPINAL MOTOR NEURONS ARE EASILY 4872 03:27:48,680 --> 03:27:48,920 TARGETED. 4873 03:27:48,920 --> 03:27:51,280 SOME OF THE EASIEST CELLS TO 4874 03:27:51,280 --> 03:28:01,760 TARGET WITH VIRAL VECTORS. 4875 03:28:05,000 --> 03:28:06,760 THAT'S A HUGE ADVANTAGE AND USE 4876 03:28:06,760 --> 03:28:12,600 OF A VECTOR TO TREAT MOTOR 4877 03:28:12,600 --> 03:28:14,280 NEURONS AND THE MOST SPECTACULAR 4878 03:28:14,280 --> 03:28:18,640 RESULT IN THE HISTORY OF GENE 4879 03:28:18,640 --> 03:28:18,840 THERAPY. 4880 03:28:18,840 --> 03:28:25,520 SO ONE QUESTION IS DO WE NEED 4881 03:28:25,520 --> 03:28:29,720 SPECIFICITY OF THE VIRAL VECTOR 4882 03:28:29,720 --> 03:28:31,760 IT DEPENDS ON WHAT YOU'RE TRYING 4883 03:28:31,760 --> 03:28:33,760 TO EXPRESS BUT WE HAVE ALSO 4884 03:28:33,760 --> 03:28:40,040 SHOWN ANOTHER CELL THAT IS 4885 03:28:40,040 --> 03:28:44,440 HIGHLY TARGETED IS THE SENSORY 4886 03:28:44,440 --> 03:28:54,200 NEURON IN THE GANGAL ROOT AND WE 4887 03:28:54,200 --> 03:28:56,600 CAN LIMIT THE EXPRESSION IN THE 4888 03:28:56,600 --> 03:28:58,680 DORSAL ROOT GANGLIA AND 4889 03:28:58,680 --> 03:29:00,480 OTHERWISE THE SPECIFICITY IS 4890 03:29:00,480 --> 03:29:06,600 DEVELOPED NOT BY TARGETING THE 4891 03:29:06,600 --> 03:29:09,440 THE CELLS AND THERE'S WAYS TO 4892 03:29:09,440 --> 03:29:11,640 ACHIEVE SUCH SPECIFICITY. 4893 03:29:11,640 --> 03:29:17,640 I WOULD SAY IT'S NOT THE RATE 4894 03:29:17,640 --> 03:29:20,080 BUT IDENTIFYING THE RIGHT PART 4895 03:29:20,080 --> 03:29:22,000 OF THE GENE WHICH SPEAKS TO THE 4896 03:29:22,000 --> 03:29:24,320 FIRST QUESTION AND FOR VIRAL 4897 03:29:24,320 --> 03:29:25,400 VECTOR IN ACHIEVING STABLE 4898 03:29:25,400 --> 03:29:27,000 EXPRESSION WE MAY HAVE CRACKED 4899 03:29:27,000 --> 03:29:27,800 THAT NUT. 4900 03:29:27,800 --> 03:29:31,240 I THINK WHAT'S NOW LIMITING ARE 4901 03:29:31,240 --> 03:29:32,680 A MODEL SYSTEM WE CAN CRANK 4902 03:29:32,680 --> 03:29:36,400 THROUGH OUR CANDIDATES WITH 4903 03:29:36,400 --> 03:29:39,280 VIRAL VECTOR IN ORDER TO 4904 03:29:39,280 --> 03:29:41,480 DETERMINE WHAT WOULD LOOK THE 4905 03:29:41,480 --> 03:29:45,680 MOST PROMISING AND THERE'S 4906 03:29:45,680 --> 03:29:46,800 ANIMAL MODELS WHERE CERTAIN 4907 03:29:46,800 --> 03:29:48,640 FORMS OF ALS THAT ARE NOT GREAT 4908 03:29:48,640 --> 03:29:52,640 AND NOT ROBUST ENOUGH TO GO INTO 4909 03:29:52,640 --> 03:29:54,040 HIGH THROUGHPUT SCREENING THAT 4910 03:29:54,040 --> 03:29:57,440 COULD BE ACHIEVED TO ADDRESS 4911 03:29:57,440 --> 03:29:58,240 THIS POTENTIAL AVENUE OF 4912 03:29:58,240 --> 03:30:07,280 RESEARCH. 4913 03:30:07,280 --> 03:30:11,680 >>THE NEXT QUESTION FOR CRATING 4914 03:30:11,680 --> 03:30:16,800 AN INFRASTRUCTURE FOR BIO SAMPLE 4915 03:30:16,800 --> 03:30:20,400 QUESTION REPOSITORYIES TO 4916 03:30:20,400 --> 03:30:22,000 UNDERSTANDING THE DISEASE AND 4917 03:30:22,000 --> 03:30:25,800 IT'S GOOD TO HAVE MEDICAL BIO 4918 03:30:25,800 --> 03:30:27,680 REPOSITORIES. 4919 03:30:27,680 --> 03:30:29,200 >>YES, IT'S SOMETHING WE 4920 03:30:29,200 --> 03:30:30,200 DISCUSSED IN THE GROUP. 4921 03:30:30,200 --> 03:30:33,800 WE HAVE NOT MADE A FIRM DECISION 4922 03:30:33,800 --> 03:30:35,360 ON THIS YET IN REGARDS TO 4923 03:30:35,360 --> 03:30:36,680 RECOMMENDATIONS BECAUSE WE WANT 4924 03:30:36,680 --> 03:30:42,920 INPUT FROM YOU ALL ALSO. 4925 03:30:42,920 --> 03:30:44,520 BUT WHERE THERE'S CENTERS ARE 4926 03:30:44,520 --> 03:30:46,760 SUBMITTING SAMPLES AND GET 4927 03:30:46,760 --> 03:30:49,720 ANALYZED TO THE BEST AVAILABLE 4928 03:30:49,720 --> 03:30:52,760 BIO MARKERS USING CENTRALIZED 4929 03:30:52,760 --> 03:30:55,200 ASSAYS LEADING TO VARIATION AND 4930 03:30:55,200 --> 03:30:58,400 THE BIO REPOSITORY WILL BE 4931 03:30:58,400 --> 03:31:00,680 POPULATED WITH DATA AND FILA 4932 03:31:00,680 --> 03:31:06,040 FILAMENTS AND OTHER BIOMARKERS 4933 03:31:06,040 --> 03:31:12,680 PROMISING FOR ALS AND RELATE 4934 03:31:12,680 --> 03:31:16,080 THEM TO ACHIEVE PROGRESS AND 4935 03:31:16,080 --> 03:31:22,200 COORDINATE FOR THE USE OF THE 4936 03:31:22,200 --> 03:31:24,640 SAMPLING SO FROM MY POINT OF 4937 03:31:24,640 --> 03:31:27,440 VIEW, BOTH ALTERNATIVES ARE 4938 03:31:27,440 --> 03:31:29,120 REALLY GREAT. 4939 03:31:29,120 --> 03:31:34,200 WE'RE FIRMLY RECOMMENDING 4940 03:31:34,200 --> 03:31:38,120 ESTABLISHING THE BIO 4941 03:31:38,120 --> 03:31:40,280 REPOSITORIES AND HAVING THEM 4942 03:31:40,280 --> 03:31:40,680 AVAILABLE. 4943 03:31:40,680 --> 03:31:41,680 I THINK WE SHOULD DISCUSS THIS A 4944 03:31:41,680 --> 03:31:48,680 LITTLE BIT. 4945 03:31:48,680 --> 03:31:51,520 >>THANK YOU. 4946 03:31:51,520 --> 03:31:59,320 NEXT QUESTION, DID YOU BELIEVE 4947 03:31:59,320 --> 03:32:01,840 THERE'S OLD FDA APPROVED DRUGS 4948 03:32:01,840 --> 03:32:02,960 THAT MAY HAVE THERAPEUTIC EFFECT 4949 03:32:02,960 --> 03:32:03,880 ON ALS THAT ARE NOT BEING 4950 03:32:03,880 --> 03:32:06,480 RECOGNIZED AND THE REPURPOSING 4951 03:32:06,480 --> 03:32:07,000 TECHNIQUE ON ALS MAY BE 4952 03:32:07,000 --> 03:32:14,640 EFFECTIVE? 4953 03:32:14,640 --> 03:32:15,880 THERE'S PROS AND CONS TO THAT 4954 03:32:15,880 --> 03:32:17,080 TYPE OF APPROACH. 4955 03:32:17,080 --> 03:32:20,080 I THINK THE POSITIVES ARE I 4956 03:32:20,080 --> 03:32:21,600 SHOWED THIS LONG DEVELOPMENT 4957 03:32:21,600 --> 03:32:23,520 TIME LINE AND USING FDA APPROVED 4958 03:32:23,520 --> 03:32:28,280 DRUGS ALLOW YOU TO SKIP A LOT OF 4959 03:32:28,280 --> 03:32:34,440 STEPS THERE. 4960 03:32:34,440 --> 03:32:35,480 THE SAFETY HAS BEEN DEMONSTRATED 4961 03:32:35,480 --> 03:32:36,840 TO YOU CAN MOVE TO LATER STAGES 4962 03:32:36,840 --> 03:32:39,960 SO THAT'S AN EXCITING APPROACH 4963 03:32:39,960 --> 03:32:41,960 AND IF WE CAN UNDERSTAND THE 4964 03:32:41,960 --> 03:32:43,240 MECHANISMS OF DISEASE AND SHOW 4965 03:32:43,240 --> 03:32:46,040 THESE DRUGS IMPACT THEM, OFTEN 4966 03:32:46,040 --> 03:32:52,280 THAT CAN BE A BIG ADVANTAGE. 4967 03:32:52,280 --> 03:32:54,520 THE DOWN SIDE TO BE REALISTIC 4968 03:32:54,520 --> 03:32:56,480 ABOUT IT, AS MENTIONED EARLIER I 4969 03:32:56,480 --> 03:32:59,400 THINK BY STEFAN THAT ONE OF THE 4970 03:32:59,400 --> 03:33:00,680 BIGGEST CHALLENGES IS DELIVERING 4971 03:33:00,680 --> 03:33:07,240 DRUGS TO THE BRAIN. 4972 03:33:07,240 --> 03:33:08,920 THEY MAY MIND AN INTERESTING 4973 03:33:08,920 --> 03:33:11,520 COMPOUND BUT THEN CORNERS ARE 4974 03:33:11,520 --> 03:33:12,600 OFTEN CUT BECAUSE THE COMPOUND 4975 03:33:12,600 --> 03:33:14,640 HAS CERTAIN QUALITIES YOU WANT. 4976 03:33:14,640 --> 03:33:17,120 YOU PUSH IT FORWARD MAYBE IF 4977 03:33:17,120 --> 03:33:20,680 IT'S NOT PERFECT FOR TREATING 4978 03:33:20,680 --> 03:33:24,680 THE DISEASE YOU'RE INTERESTED 4979 03:33:24,680 --> 03:33:26,280 IN. 4980 03:33:26,280 --> 03:33:28,280 THAT'S SOMETHING WE NEED TO BE 4981 03:33:28,280 --> 03:33:35,960 CAREFUL OF OF BRYNING -- 4982 03:33:35,960 --> 03:33:36,720 BRINGING THE COMPOUND FORWARD 4983 03:33:36,720 --> 03:33:38,320 AND WHETHER THEY'RE USED 4984 03:33:38,320 --> 03:33:39,800 DIRECTLY OR USED AS TOOLS FOR 4985 03:33:39,800 --> 03:33:41,640 NEXT GENERATION OF MEDICINES, I 4986 03:33:41,640 --> 03:33:43,600 THINK IT CAN BE AN EFFECTIVE 4987 03:33:43,600 --> 03:33:49,640 STRATEGY. 4988 03:33:49,640 --> 03:33:52,760 >>GREAT, THANKS. 4989 03:33:52,760 --> 03:33:53,280 HENRIK, WE HAVE ANOTHER 4990 03:33:53,280 --> 03:33:54,480 BIOMARKER QUESTION FOR YOU, 4991 03:33:54,480 --> 03:33:57,080 WHICH IS WHAT KIND OF NUMBERS IN 4992 03:33:57,080 --> 03:33:58,000 TERMS PATIENT PARTICIPATION WILL 4993 03:33:58,000 --> 03:33:59,920 BE NEEDED TO ADDRESS YOUR 4994 03:33:59,920 --> 03:34:02,640 PRIORITIES AND IN PARTICULAR TO 4995 03:34:02,640 --> 03:34:04,120 VALIDATE A BIOMARKER OF DISEASE 4996 03:34:04,120 --> 03:34:04,400 PROGRESSION. 4997 03:34:04,400 --> 03:34:08,920 WE'VE TALKED ABOUT THAT A LOT. 4998 03:34:08,920 --> 03:34:11,280 IT WOULD BE PRESUMABLE TO THE 4999 03:34:11,280 --> 03:34:13,880 FDA OPPOSED TO A NICE 5000 03:34:13,880 --> 03:34:15,520 PEER-REVIEWED PAPER PUBLICATION. 5001 03:34:15,520 --> 03:34:16,640 I THINK TALKING A LITTLE BIT 5002 03:34:16,640 --> 03:34:18,640 ABOUT WHAT NUMBERS DO WE NEED 5003 03:34:18,640 --> 03:34:20,680 AND AS WE'RE TALKING ABOUT 5004 03:34:20,680 --> 03:34:22,480 DIFFERENT SUB TYPES OF PATIENTS 5005 03:34:22,480 --> 03:34:26,200 WHAT NUMBERS DO WE NEED TO START 5006 03:34:26,200 --> 03:34:36,640 TO PLOT MEANINGFUL THINGS? 5007 03:34:44,160 --> 03:34:47,600 >>THERE'S PROCESSES AT PLAY. 5008 03:34:47,600 --> 03:34:52,840 IT DEPENDS ON HOW STRONGLY THE 5009 03:34:52,840 --> 03:34:55,040 AND IT'S THE EFFECT SIZE OF THE 5010 03:34:55,040 --> 03:35:00,200 BIOMARKER THAT WILL SHOW THIS 5011 03:35:00,200 --> 03:35:10,680 AND YOU CAN SEE AND THERE'S 5012 03:35:12,720 --> 03:35:22,960 DYNAMICS IN IT. 5013 03:35:27,560 --> 03:35:29,560 AND THEY ALSO SEE WE DON'T NEED 5014 03:35:29,560 --> 03:35:31,480 HIGH NUMBERS BUT WE NEED HIGH 5015 03:35:31,480 --> 03:35:36,680 NUMBERS IF WE'RE TO STUDY 5016 03:35:36,680 --> 03:35:41,720 APPROACHES AND THE CHANGES NEED 5017 03:35:41,720 --> 03:35:43,000 TO BE SMALLER. 5018 03:35:43,000 --> 03:35:44,200 THIS COULD BE A GLOBAL 5019 03:35:44,200 --> 03:35:45,520 COORDINATION TO INCREASE THE 5020 03:35:45,520 --> 03:35:50,560 NUMBERS SINCE THERE'S MANY TYPES 5021 03:35:50,560 --> 03:35:52,760 OF ALS AND THE NUMBERS THE MORE 5022 03:35:52,760 --> 03:36:03,000 THE BETTER. 5023 03:36:05,440 --> 03:36:06,480 IT'S IMPORTANT WE COLLABORATE 5024 03:36:06,480 --> 03:36:09,040 AND SHARE THE RESOURCES. 5025 03:36:09,040 --> 03:36:12,680 I'M PRETTY SURE WE'LL SEE 5026 03:36:12,680 --> 03:36:23,200 INTERESTING THINGS COMING UP AND 5027 03:36:24,120 --> 03:36:28,680 LOOK AT CHEMICAL BIOMARKERS. 5028 03:36:28,680 --> 03:36:33,320 IF THEY WANT PROSPECTUS STUDIES 5029 03:36:33,320 --> 03:36:34,560 THAT'S IMPORTANT AND YOU CAN 5030 03:36:34,560 --> 03:36:36,120 TOGETHER DESIGN A PROSPECTUS 5031 03:36:36,120 --> 03:36:38,320 STUDY TO EVALUATE HOW WELL A 5032 03:36:38,320 --> 03:36:39,640 BIOMARKER WORKS TO TRY DISEASE 5033 03:36:39,640 --> 03:36:50,320 OR PREDICT DISEASE PROGRESSION 5034 03:36:52,920 --> 03:36:58,200 AND WE MAY GET TO KNOW EACH 5035 03:36:58,200 --> 03:36:59,440 OTHER AND WE COULD DESCRIBE THE 5036 03:36:59,440 --> 03:37:04,800 BIOMARKER AND I WOULD LIKE TO 5037 03:37:04,800 --> 03:37:15,120 VALIDATE THE STUDY 5038 03:37:19,160 --> 03:37:22,320 >>PLEASE COMMENT ON POTENTIAL 5039 03:37:22,320 --> 03:37:25,400 NEED TO DEVELOP ALS APPROACHES 5040 03:37:25,400 --> 03:37:27,160 TO TARGET THE ALLELE 5041 03:37:27,160 --> 03:37:27,560 SPECIFICALLY. 5042 03:37:27,560 --> 03:37:30,280 IS IT ADVERSE TO SUPPRESS THE 5043 03:37:30,280 --> 03:37:34,240 MUTANT AND NON-MUTANT ALLELE. 5044 03:37:34,240 --> 03:37:35,800 THIS IS AN IMPORTANT QUESTION. 5045 03:37:35,800 --> 03:37:44,040 I THINK IT RAISES A POINT THAT 5046 03:37:44,040 --> 03:37:47,160 WAS MADE EARLIER BY JIM THAT THE 5047 03:37:47,160 --> 03:37:50,040 ALS INVESTIGATORS CLINICIANS AND 5048 03:37:50,040 --> 03:37:51,280 SCIENTISTS, HAVE THE OPPORTUNITY 5049 03:37:51,280 --> 03:37:55,800 NOW GIVEN EXISTING TECHNOLOGIES 5050 03:37:55,800 --> 03:38:05,880 TO DEVELOP GENE-BASED 5051 03:38:05,880 --> 03:38:08,160 THERAPEUTICS AND INTRODUCE THEM 5052 03:38:08,160 --> 03:38:10,320 WITH LITTLE OR NO INDUSTRY 5053 03:38:10,320 --> 03:38:14,120 SUPPORT. 5054 03:38:14,120 --> 03:38:24,600 IN SOME CASES AND SOME ASOs 5055 03:38:30,680 --> 03:38:32,280 TARGET ALL TRANSCRIPTS OF THE 5056 03:38:32,280 --> 03:38:35,200 GENES AND THE THINKING IS YOU 5057 03:38:35,200 --> 03:38:37,320 CAN DO IT SAFELY. 5058 03:38:37,320 --> 03:38:38,960 THERE'S OTHER WHERE DEFICIENCY 5059 03:38:38,960 --> 03:38:40,800 STATES CANNOT BE TOLERATED AND 5060 03:38:40,800 --> 03:38:46,320 IT WOULD BE DANGEROUS TO KNOCK 5061 03:38:46,320 --> 03:38:50,560 DOWN A GENE IRRESPECTIVE OF THE 5062 03:38:50,560 --> 03:38:51,640 MUTATION OR NOT. 5063 03:38:51,640 --> 03:38:54,040 IN THOSE CASES YOU'LL NEED TO GO 5064 03:38:54,040 --> 03:38:56,680 AFTER THE MUTANT ALLELE AND COPY 5065 03:38:56,680 --> 03:39:06,080 IN A SPECIFIC WAY. 5066 03:39:06,080 --> 03:39:09,760 AND DR. BROWN AT U-MASS IS DOING 5067 03:39:09,760 --> 03:39:14,000 THAT AND WE'RE DOING THAT 5068 03:39:14,000 --> 03:39:15,160 THROUGH THE SCIENCE ALS 5069 03:39:15,160 --> 03:39:15,760 INITIATIVE AND THESE WILL BE 5070 03:39:15,760 --> 03:39:25,520 RARE CASES. 5071 03:39:25,520 --> 03:39:27,720 AND THIS IS GOING TO NEED TO 5072 03:39:27,720 --> 03:39:32,760 HAPPEN IN THE NON-PROFIT SPACE 5073 03:39:32,760 --> 03:39:35,240 AND HERE'S WHERE I THINK NIH AND 5074 03:39:35,240 --> 03:39:37,240 PUBLIC SUPPORT GENERALLY WILL BE 5075 03:39:37,240 --> 03:39:40,320 NEEDED TO MOVE THE THERAPEUTICS 5076 03:39:40,320 --> 03:39:43,480 THROUGH IN SOME CASES IN AN 5077 03:39:43,480 --> 03:39:45,640 INDIVIDUAL WAY OR IN SMALL 5078 03:39:45,640 --> 03:39:48,080 POPULATIONS AND THAT IS EXCITING 5079 03:39:48,080 --> 03:39:55,600 OPPORTUNITY I THINK FOR US. 5080 03:39:55,600 --> 03:39:58,280 >>IT'S WELL STATED. 5081 03:39:58,280 --> 03:40:04,120 THE OVER ALL ISSUE IS IT'S AN 5082 03:40:04,120 --> 03:40:08,480 ELEGANT CONCEPTUAL APPROACH AND 5083 03:40:08,480 --> 03:40:12,520 SPARING THE NON-DISEASE COPY OF 5084 03:40:12,520 --> 03:40:16,080 THE ALLELE AND IF THE GENE IS 5085 03:40:16,080 --> 03:40:17,800 CRUCIAL, YES, ABSOLUTELY. 5086 03:40:17,800 --> 03:40:21,440 IF IT'S NOT CRUCIAL, NO, YOU 5087 03:40:21,440 --> 03:40:22,000 DON'T NEED TO. 5088 03:40:22,000 --> 03:40:25,360 MOST FALL IN BETWEEN THE GRAY 5089 03:40:25,360 --> 03:40:28,640 MATTER WHERE WE DON'T KNOW 5090 03:40:28,640 --> 03:40:32,520 ENOUGH AND HAVE TO TRY TO INFER 5091 03:40:32,520 --> 03:40:33,880 FROM MODEL AND ANIMAL 5092 03:40:33,880 --> 03:40:34,560 INFORMATION AND WHAT'S THE 5093 03:40:34,560 --> 03:40:36,680 RETURN FOR WHAT CAN BE A STEEP 5094 03:40:36,680 --> 03:40:47,080 ENGINEERING CHALLENGE. 5095 03:40:49,440 --> 03:40:51,320 >>THIS IS SOMETHING SOME CARE 5096 03:40:51,320 --> 03:40:52,960 TREMENDOUSLY IN THE FIELD ABOUT 5097 03:40:52,960 --> 03:41:02,480 WHO GETS PRIORITIZED FOR TRIALS. 5098 03:41:02,480 --> 03:41:12,880 AND THOSE THAT ARE P 5099 03:41:16,600 --> 03:41:17,320 PRESYMPTOMATIC TRIALS. 5100 03:41:17,320 --> 03:41:27,760 IT'S A CRITICAL QUESTION. 5101 03:41:33,240 --> 03:41:36,240 >>WE NEED TO UNDERSTAND WHEN 5102 03:41:36,240 --> 03:41:37,960 THE ALS PATHOLOGY STARTS AND WHY 5103 03:41:37,960 --> 03:41:39,200 IT STARTS IN SOME PEOPLE AND NOT 5104 03:41:39,200 --> 03:41:44,240 IN OTHERS. 5105 03:41:44,240 --> 03:41:50,160 ALL THOSE THINGS WILL BE BETTER 5106 03:41:50,160 --> 03:41:51,560 UNDERSTOOD IF MORE PARTICIPATE 5107 03:41:51,560 --> 03:41:54,080 IN THE GENE THERAPY STUDIES. 5108 03:41:54,080 --> 03:41:55,240 IN ACCESSING TREATMENT EARLIER, 5109 03:41:55,240 --> 03:41:59,600 YES, TREATMENTS SHOULD BE 5110 03:41:59,600 --> 03:42:02,600 ACCESSED EARLY WHEN WE KNOW THAT 5111 03:42:02,600 --> 03:42:03,800 THOSE TREATMENTS ARE NOT GOING 5112 03:42:03,800 --> 03:42:06,040 TIP SOMEBODY OVER TO THE DISEASE 5113 03:42:06,040 --> 03:42:06,720 PROCESS WHEN THERE ARE NOT A LOT 5114 03:42:06,720 --> 03:42:09,160 OF SIDE EFFECTS TO DEAL WITH. 5115 03:42:09,160 --> 03:42:13,080 SO CURRENTLY APPROVED TREATMENTS 5116 03:42:13,080 --> 03:42:15,080 DO NOT HAVE A LOT OF SIDE 5117 03:42:15,080 --> 03:42:18,400 EFFECTS SO I SEE WHY SOMEONE WHO 5118 03:42:18,400 --> 03:42:22,120 IS PRESYMPTOMATIC WOULD WANT TO 5119 03:42:22,120 --> 03:42:24,320 TRI THESE. 5120 03:42:24,320 --> 03:42:29,600 THAT NEEDS TO BE STUDIED IN 5121 03:42:29,600 --> 03:42:32,080 PEOPLE WHO ARE PRESYMPTOMATIC 5122 03:42:32,080 --> 03:42:34,200 INDIVIDUALS AND WHAT'S THE 5123 03:42:34,200 --> 03:42:34,440 TREATMENT? 5124 03:42:34,440 --> 03:42:37,000 IT'S KNOWING WHEN IS THE RIGHT 5125 03:42:37,000 --> 03:42:39,640 TIME TO START DOSING SOMEBODY. 5126 03:42:39,640 --> 03:42:41,600 WHEN YOU SEE ALS LOOMING IN YOUR 5127 03:42:41,600 --> 03:42:43,200 FUTURE, YOU WANT TO TRY 5128 03:42:43,200 --> 03:42:43,560 ANYTHING. 5129 03:42:43,560 --> 03:42:44,680 YOU WANT TO TRY ANYTHING TO MAKE 5130 03:42:44,680 --> 03:42:47,680 THAT STOP. 5131 03:42:47,680 --> 03:42:50,400 PEOPLE SAY THEY'RE NOT WORRIED 5132 03:42:50,400 --> 03:42:54,520 ABOUT SIDE EFFECTS BUT THERE'S A 5133 03:42:54,520 --> 03:42:56,400 REAL CAUTION WE HAVE TO HAVE AND 5134 03:42:56,400 --> 03:42:57,760 APPROACH THIS CAREFULLY BECAUSE 5135 03:42:57,760 --> 03:42:59,720 WE DON'T WANT TO TIP SOMEBODY 5136 03:42:59,720 --> 03:43:04,680 OVER INTO THE DISEASE EARLIER. 5137 03:43:04,680 --> 03:43:07,600 THERE IS NOTHING WORSE THAN 5138 03:43:07,600 --> 03:43:09,320 HAVING TO MANAGE MORE SERIOUS 5139 03:43:09,320 --> 03:43:10,680 SIDE EFFECTS ON TOP OF THE 5140 03:43:10,680 --> 03:43:11,560 WORRIES OF ALS. 5141 03:43:11,560 --> 03:43:15,440 I JUST THINK IT NEEDS A CAREFUL 5142 03:43:15,440 --> 03:43:16,320 APPROACH AND BIOMARKERS WILL BE 5143 03:43:16,320 --> 03:43:18,240 THE WAY TO GET THERE. 5144 03:43:18,240 --> 03:43:20,720 I WELCOME ANY OTHER RESPONSES 5145 03:43:20,720 --> 03:43:31,160 FROM PEOPLE ON THIS TEAM. 5146 03:43:32,880 --> 03:43:34,960 AND PEOPLE WHO ARE GENE CARE REZ 5147 03:43:34,960 --> 03:43:36,960 HAVE A HIGHER THAN NORMAL RISK 5148 03:43:36,960 --> 03:43:46,800 TOLERANCE BUT IT'S NOT THE SAME. 5149 03:43:46,800 --> 03:43:50,800 AND WHAT ARE THE POTENTIAL 5150 03:43:50,800 --> 03:43:52,680 CONSEQUENCES OF LONG-TERM 5151 03:43:52,680 --> 03:43:53,840 TREATMENT WITH SAY AN EXAMPLE A 5152 03:43:53,840 --> 03:43:54,160 THERAPEUTIC. 5153 03:43:54,160 --> 03:44:02,400 WE DON'T KNOW. 5154 03:44:02,400 --> 03:44:03,640 AND THERE'S REASONS TO BE MORE 5155 03:44:03,640 --> 03:44:04,280 CAUTIOUS AS NADIA IS POINTING 5156 03:44:04,280 --> 03:44:14,360 OUT. 5157 03:44:16,400 --> 03:44:17,600 AND IF YOU'RE TEAM GOT 5158 03:44:17,600 --> 03:44:19,000 EVERYTHING YOU IDENTIFIED AS 5159 03:44:19,000 --> 03:44:22,080 CRITICAL FOR IMPROVING, HOW 5160 03:44:22,080 --> 03:44:23,320 WOULD AFFECT THE 15-YEAR TIME 5161 03:44:23,320 --> 03:44:28,320 LINE FOR DRUG DEVELOPMENT AND 5162 03:44:28,320 --> 03:44:33,520 LESS THAN 5% SUCCESS RATE 5163 03:44:33,520 --> 03:44:35,040 >>I SAW THE QUESTION COME 5164 03:44:35,040 --> 03:44:35,920 THROUGH THE Q&A DURING THE 5165 03:44:35,920 --> 03:44:36,480 SESSION AND IS PARTICULARLY 5166 03:44:36,480 --> 03:44:46,640 IMPORTANT. 5167 03:44:50,160 --> 03:44:52,120 I SHARED THE REALITY IN TERMS OF 5168 03:44:52,120 --> 03:44:54,520 PIPELINE AND POTENTIAL FOR 5169 03:44:54,520 --> 03:44:54,760 SUCCESS. 5170 03:44:54,760 --> 03:44:57,000 I THINK THIS IS A CORE QUESTION 5171 03:44:57,000 --> 03:44:58,240 OF HOW MUCH IS THIS GOING TO 5172 03:44:58,240 --> 03:45:07,920 IMPACT THINGS IN A POSITIVE 5173 03:45:07,920 --> 03:45:08,240 DIRECTION. 5174 03:45:08,240 --> 03:45:11,360 AND THE IMPACT OF THESE TYPES OF 5175 03:45:11,360 --> 03:45:12,640 EFFORTS CAN BE SUBSTANTIAL BOTH 5176 03:45:12,640 --> 03:45:15,560 IN TERMS OF TIME LINES AND 5177 03:45:15,560 --> 03:45:17,360 POTENTIAL FOR SUCCESS AND I'D 5178 03:45:17,360 --> 03:45:19,080 LOVE TO HEAR COMMENTS FROM 5179 03:45:19,080 --> 03:45:20,040 OTHERS ON THE PANEL AS WELL. 5180 03:45:20,040 --> 03:45:25,800 BUT IN TERMS OF TIME LINES, I 5181 03:45:25,800 --> 03:45:28,320 THINK JIM HIGHLIGHTED NEW 5182 03:45:28,320 --> 03:45:29,640 THERAPEUTIC APPROACHES THAT ARE 5183 03:45:29,640 --> 03:45:32,640 TARGETED AND HAVE SHORTER TIME 5184 03:45:32,640 --> 03:45:32,880 LINES. 5185 03:45:32,880 --> 03:45:34,360 THOSE ARE STILL NOT APPLICABLE 5186 03:45:34,360 --> 03:45:37,160 TO THE MAJORITY OF ALSES BASED 5187 03:45:37,160 --> 03:45:38,720 ON WHAT WE KNOW ABOUT DISEASE. 5188 03:45:38,720 --> 03:45:40,640 SOME OF THE THINGS WE'RE TRYING 5189 03:45:40,640 --> 03:45:42,920 TO ESTABLISH ARE TO GENERATE 5190 03:45:42,920 --> 03:45:43,920 THAT UNDERSTANDING. 5191 03:45:43,920 --> 03:45:46,240 AND THEN IN TERMS OF POTENTIAL 5192 03:45:46,240 --> 03:45:48,160 FOR SUCCESS, I THINK THE IMPACT 5193 03:45:48,160 --> 03:45:52,080 IS PERHAPS EVEN MORE TANGIBLE 5194 03:45:52,080 --> 03:45:53,320 WHERE BETTER UNDERSTANDING 5195 03:45:53,320 --> 03:45:56,520 DISEASE TO MATCH THE RIGHT 5196 03:45:56,520 --> 03:45:58,320 PATIENTS WITH THE RIGHT DRUGS I 5197 03:45:58,320 --> 03:46:00,320 THINK CAN HAVE AN ORDER OF 5198 03:46:00,320 --> 03:46:03,040 MAGNITUDE LEAP IN TERMS OF OUR 5199 03:46:03,040 --> 03:46:05,520 SUCCESS BRINGING DRUGS FORWARD. 5200 03:46:05,520 --> 03:46:07,800 THUS FAR I THINK THE SUCCESS HAS 5201 03:46:07,800 --> 03:46:09,840 BEEN VERY LIMITED AND REALLY TO 5202 03:46:09,840 --> 03:46:12,520 GET CLOSER TO THE CURE TO HAVE A 5203 03:46:12,520 --> 03:46:14,120 BIG IMPACT WE NEED TO 5204 03:46:14,120 --> 03:46:15,080 FUNDAMENTALLY IMPROVE THAT 5205 03:46:15,080 --> 03:46:16,320 UNDERSTANDING AND MAKING SURE 5206 03:46:16,320 --> 03:46:19,720 WE'RE MATCHING DRUGS WITH 5207 03:46:19,720 --> 03:46:19,960 PATIENTS. 5208 03:46:19,960 --> 03:46:25,280 SO I'D LOVE TO IT UP TO OTHERS 5209 03:46:25,280 --> 03:46:26,240 FOR COMMENTS BECAUSE I THINK 5210 03:46:26,240 --> 03:46:28,320 THAT'S THE REASON WE'RE ALL HERE 5211 03:46:28,320 --> 03:46:31,040 TODAY TO SOME DEGREE. 5212 03:46:31,040 --> 03:46:31,400 >>YEAH. 5213 03:46:31,400 --> 03:46:34,360 IT DEPENDS ON WHAT WE FIND. 5214 03:46:34,360 --> 03:46:36,680 THE BEST CASE SCENARIO WE FIND 5215 03:46:36,680 --> 03:46:40,320 AS LDL IS TO HEART DISEASE, X IS 5216 03:46:40,320 --> 03:46:44,680 TO ALS. 5217 03:46:44,680 --> 03:46:48,680 WE WON'T KNOW UNTIL WE HAVE A 5218 03:46:48,680 --> 03:46:49,640 GOOD LOOK AT IT. 5219 03:46:49,640 --> 03:46:54,360 >>WE STARTED OFF WITH 5220 03:46:54,360 --> 03:46:56,320 BIOMARKERS BEING FUNDAMENTAL. 5221 03:46:56,320 --> 03:46:58,360 I'LL THANK EVERYBODY AND THANK 5222 03:46:58,360 --> 03:47:01,200 MY FELLOW MEMBERS OF THIS 5223 03:47:01,200 --> 03:47:04,440 WORKING GROUP AND THE AUDIENCE 5224 03:47:04,440 --> 03:47:06,400 FOR TERRIFIC QUESTIONS. 5225 03:47:06,400 --> 03:47:08,840 IT'S BEEN A GREAT PROCESS IN 5226 03:47:08,840 --> 03:47:10,400 THINKING THROUGH HOW CAN WE HELP 5227 03:47:10,400 --> 03:47:13,640 MAKE A DIFFERENCE IN THE FIELD. 5228 03:47:13,640 --> 03:47:14,600 NEIL, ANYTHING TO ADD? 5229 03:47:14,600 --> 03:47:16,160 >>I WANT TO ADD MY THANKS TO 5230 03:47:16,160 --> 03:47:21,960 THE GROUP. 5231 03:47:21,960 --> 03:47:24,080 WORKING WITH THESE WONDERFULLY 5232 03:47:24,080 --> 03:47:25,120 THOUGHTFUL PEOPLE CAN'T HELP BUT 5233 03:47:25,120 --> 03:47:26,920 LEAVE ME OPTIMISTIC WE'LL MAKE 5234 03:47:26,920 --> 03:47:28,360 REAL ADVANCES IN THE YEARS TO 5235 03:47:28,360 --> 03:47:31,480 COME AND THANKS TO DR. KOROSHETZ 5236 03:47:31,480 --> 03:47:33,320 AND THE NIH LEADERSHIP FOR THIS 5237 03:47:33,320 --> 03:47:35,200 REALLY IMPORTANT INITIATIVE AND 5238 03:47:35,200 --> 03:47:40,360 FOR THE STEERING COMMITTEE AND A 5239 03:47:40,360 --> 03:47:43,600 SPECIAL THANKS TO EMILY WHO LED 5240 03:47:43,600 --> 03:47:44,520 US THROUGHOUT THIS. 5241 03:47:44,520 --> 03:47:47,280 THANK YOU, EMILY. 5242 03:47:47,280 --> 03:47:50,720 >>THANK YOU, EVERYONE. 5243 03:47:50,720 --> 03:47:52,320 NOW WE'RE READY TO GO TO BREAK 5244 03:47:52,320 --> 03:47:53,680 AGAIN, IS THAT RIGHT? 5245 03:47:53,680 --> 03:47:53,960 RITA? 5246 03:47:53,960 --> 03:47:55,440 >>THANK YOU, EVERYBODY. 5247 03:47:55,440 --> 03:47:56,840 THANK YOU, NEIL AND STACIE AND 5248 03:47:56,840 --> 03:47:58,760 THE REST OF THE WORKING GROUP 5249 03:47:58,760 --> 03:47:59,920 MEMBERS. 5250 03:47:59,920 --> 03:48:02,360 THIS WAS A TERRIFIC SESSION. 5251 03:48:02,360 --> 03:48:03,520 WONDERFUL QUESTIONS FROM THE 5252 03:48:03,520 --> 03:48:03,760 AUDIENCE. 5253 03:48:03,760 --> 03:48:07,800 WE'RE EXCITED WE HAVE SUCH A 5254 03:48:07,800 --> 03:48:09,200 GREAT COMMUNICATION AND 5255 03:48:09,200 --> 03:48:10,200 PARTICIPATION IN THIS. 5256 03:48:10,200 --> 03:48:13,600 WE'LL TAKE A 15-MINUTE BREAK SO 5257 03:48:13,600 --> 03:48:15,440 WE'LL BE BACK AT 2:30 EASTERN 5258 03:48:15,440 --> 03:48:18,040 WHATEVER TIME THAT IS IN YOUR 5259 03:48:18,040 --> 03:48:19,640 OWN TIME ZONE AND WE'LL CONTINUE 5260 03:48:19,640 --> 03:48:21,600 FROM THERE WITH OUR LAST WORKING 5261 03:48:21,600 --> 03:48:22,360 GROUP DISCUSSION. 5262 03:48:22,360 --> 03:48:25,360 THANKS, EVERYBODY AND I'LL SEE 5263 03:48:25,360 --> 03:48:28,200 YOU IN 15 MINUTES 5264 03:48:28,200 --> 03:48:36,320 >>WELCOME BACK EVERYONE. 5265 03:48:36,320 --> 03:48:37,760 I'M DELIGHTED TO TURN THE 5266 03:48:37,760 --> 03:48:41,600 PROGRAM TO THE CO-CHAIRS, MERIT 5267 03:48:41,600 --> 03:48:45,400 CUDKOWICZ AND TIMOTHY MILLER 5268 03:48:45,400 --> 03:48:48,400 MANAGING THE DISCUSSION ON 5269 03:48:48,400 --> 03:48:53,280 OPTIMIZING ALS CLINICAL 5270 03:48:53,280 --> 03:48:53,600 RESEARCH. 5271 03:48:53,600 --> 03:48:55,680 >>WE WANT TO WELCOME THE OTHER 5272 03:48:55,680 --> 03:48:57,160 MEMBERS OF THE WORKING GROUP TO 5273 03:48:57,160 --> 03:49:00,240 TURN ON THEIR CAMERAS AND IF YOU 5274 03:49:00,240 --> 03:49:01,800 HAVEN'T, TURN IT ON NOW. 5275 03:49:01,800 --> 03:49:03,040 WE'LL INTRODUCE OURSELVES AND 5276 03:49:03,040 --> 03:49:03,880 START WITH A SHORT PRESENTATION 5277 03:49:03,880 --> 03:49:05,480 AND I THINK HAVE LOTS OF TIME 5278 03:49:05,480 --> 03:49:06,000 FOR QUESTIONS. 5279 03:49:06,000 --> 03:49:11,120 I'LL START WITH INTRODUCING 5280 03:49:11,120 --> 03:49:11,360 MYSELF. 5281 03:49:11,360 --> 03:49:16,880 I'M TILL MILLER A NEUROSCIENTIST 5282 03:49:16,880 --> 03:49:19,760 AT WASHINGTON UNIVERSITY AND 5283 03:49:19,760 --> 03:49:21,480 UNDERSTANDING AT DEVELOPING 5284 03:49:21,480 --> 03:49:24,480 NOVEL THERAPEUTIC APPROACHES AND 5285 03:49:24,480 --> 03:49:32,320 UNDERSTANDING MECHANISMS. 5286 03:49:32,320 --> 03:49:32,480 BOB. 5287 03:49:32,480 --> 03:49:36,840 >>BOB BOWSER AT THE BARO 5288 03:49:36,840 --> 03:49:37,400 NEUROLOGICAL INSTITUTE IN 5289 03:49:37,400 --> 03:49:38,440 PHOENIX, ARIZONA. 5290 03:49:38,440 --> 03:49:39,840 I WORK ON BIOMARKERS IN THE 5291 03:49:39,840 --> 03:49:41,640 DISEASE AND MOLECULAR MECHANISMS 5292 03:49:41,640 --> 03:49:46,720 AND TRANSLATIONAL NEUROSCIENCE. 5293 03:49:46,720 --> 03:49:52,640 >>I'M CHRISTIE FOURNIER AND 5294 03:49:52,640 --> 03:49:54,200 NEUROLOGIST AND CLINICAL 5295 03:49:54,200 --> 03:49:56,680 RESEARCHER WITH AN INTEREST IN 5296 03:49:56,680 --> 03:50:01,680 IMPROVING OUTCOME MEASURES FOR 5297 03:50:01,680 --> 03:50:01,840 ALS. 5298 03:50:01,840 --> 03:50:09,760 >>I'M CHIADDI ONYLKE A NEURO 5299 03:50:09,760 --> 03:50:13,360 PSYCHOLOGIST AT JOHNS HOPKINS 5300 03:50:13,360 --> 03:50:17,600 AND UNDERSTANDING THE OVERLAP 5301 03:50:17,600 --> 03:50:20,160 WITH ALS AND TREATMENT AND 5302 03:50:20,160 --> 03:50:21,960 DEVELOPMENT AND CLINICAL TRIALS. 5303 03:50:21,960 --> 03:50:26,880 >>I'M GWEN PETERSEN OF. 5304 03:50:26,880 --> 03:50:31,240 I WAS DIAGNOSED WITH ALS IN 2018 5305 03:50:31,240 --> 03:50:37,800 THANKS FOR HAVING ME. 5306 03:50:37,800 --> 03:50:44,200 >>HELLO, MELANIE QUINTANA AT 5307 03:50:44,200 --> 03:50:47,600 BERRY CONSULTANTS WHERE WE 5308 03:50:47,600 --> 03:50:48,560 DESIGN INNOVATIVE AND CLINICAL 5309 03:50:48,560 --> 03:50:50,040 TRIALS TRIALS ACROSS A WIDE 5310 03:50:50,040 --> 03:50:52,200 RANGE OF THERAPEUTIC AREAS IN 5311 03:50:52,200 --> 03:50:57,040 PARTICULAR INTERESTED IN 5312 03:50:57,040 --> 03:50:59,600 DEVELOPING DISEASE PROGRESSION 5313 03:50:59,600 --> 03:51:02,400 MODELS IN SEVERAL DISEASES. 5314 03:51:02,400 --> 03:51:05,640 >>I'M JEREMY SCHEFNER FROM THE 5315 03:51:05,640 --> 03:51:08,120 BARO NEUROLOGICAL INSTITUTE IN 5316 03:51:08,120 --> 03:51:10,160 PHOENIX AND CLINIC NEUROLOGIST 5317 03:51:10,160 --> 03:51:11,480 AND INTERESTED IN DESIGNING 5318 03:51:11,480 --> 03:51:12,840 CLINICAL TRIALS FOR ALS MANY 5319 03:51:12,840 --> 03:51:13,600 YEARS AND HAVE SPECIFIC INTEREST 5320 03:51:13,600 --> 03:51:19,840 IN DEVELOPING OUTCOME MEASURES. 5321 03:51:19,840 --> 03:51:21,800 I KNOW STEVEN GOUTMAN IS NOT 5322 03:51:21,800 --> 03:51:25,760 ABLE TO JOIN US THE PROFESSOR OF 5323 03:51:25,760 --> 03:51:27,680 NEUROLOGY AT THE UNIVERSITY OF 5324 03:51:27,680 --> 03:51:32,600 MICHIGAN AND NOT SURE IF ALBERTO 5325 03:51:32,600 --> 03:51:33,800 IS ON NORT. 5326 03:51:33,800 --> 03:51:37,200 -- ON OR NOT. 5327 03:51:37,200 --> 03:51:40,120 ALBERTO ASCHERIO AT HARVARD 5328 03:51:40,120 --> 03:51:41,080 SCHOOL OF PUBLIC HEALTH. 5329 03:51:41,080 --> 03:51:42,480 >>HE'S NOT HERE AT THE MOMENT. 5330 03:51:42,480 --> 03:51:43,080 >>OKAY. 5331 03:51:43,080 --> 03:51:43,520 VERY GOOD. 5332 03:51:43,520 --> 03:51:45,600 I'M DELIGHTED TO BE HERE AND TO 5333 03:51:45,600 --> 03:51:47,280 BE WORKING WITH THIS GROUP. 5334 03:51:47,280 --> 03:51:51,720 THIS WAS A LOT OF FUN TO GO 5335 03:51:51,720 --> 03:51:53,560 THROUGH THE PROCESS I'LL NOW 5336 03:51:53,560 --> 03:51:56,520 HAND IT OVER TO MERIT TO KICK US 5337 03:51:56,520 --> 03:51:56,920 OFF. 5338 03:51:56,920 --> 03:52:04,000 >>I'M MERIT CUDKOWICZ CHAIR OF 5339 03:52:04,000 --> 03:52:07,120 NEUROLOGY AT HARVARD MEDICAL 5340 03:52:07,120 --> 03:52:10,480 SCHOOL AND WITH JEREMY IN 1995 5341 03:52:10,480 --> 03:52:12,840 WAS HONORED TO START THE ALS 5342 03:52:12,840 --> 03:52:15,440 CONSORTIUM AND HAVE FOCUSSED ON 5343 03:52:15,440 --> 03:52:17,320 IMPROVING CLINICAL TRIALS AND 5344 03:52:17,320 --> 03:52:19,280 RESEARCH IN THE FIELD AND 5345 03:52:19,280 --> 03:52:20,280 EXCITED TO HAVE WORKED WITH TIM 5346 03:52:20,280 --> 03:52:25,600 AND OTHERS ON THIS WORKING GROUP 5347 03:52:25,600 --> 03:52:25,840 SESSION. 5348 03:52:25,840 --> 03:52:27,520 OUR GROUP MET SEVERAL TIMES AND 5349 03:52:27,520 --> 03:52:33,760 OUR PRIORITIES ARE VERY 5350 03:52:33,760 --> 03:52:34,760 INTRAREINT 5351 03:52:34,760 --> 03:52:37,600 INTRARELATE AND THINKING OF GAPS 5352 03:52:37,600 --> 03:52:38,520 IN CLINICAL TRIAL WE COULD SOLVE 5353 03:52:38,520 --> 03:52:39,920 WOULD SPEED UP DEVELOPMENT. 5354 03:52:39,920 --> 03:52:42,320 WE HEARD IN THE PREVIOUS MORNING 5355 03:52:42,320 --> 03:52:44,640 AND EARLY AFTERNOON ABOUT SOME 5356 03:52:44,640 --> 03:52:46,520 OF THE GREAT EXCITEMENT COMING 5357 03:52:46,520 --> 03:52:49,640 FROM SCIENCE AND TRANSLATION AND 5358 03:52:49,640 --> 03:52:50,600 THE GAPS THERE. 5359 03:52:50,600 --> 03:52:52,240 AND PREFACE BY SAYING THE MORE 5360 03:52:52,240 --> 03:52:53,640 WE UNDERSTAND WITH THE SCIENCE 5361 03:52:53,640 --> 03:52:55,880 THE BETTER OUR TREATMENTS AND 5362 03:52:55,880 --> 03:52:57,640 TARGETS ARE GOING TO BE FOR 5363 03:52:57,640 --> 03:53:03,440 BRINGING THEM TO PATIENTS BUT 5364 03:53:03,440 --> 03:53:05,320 THERE'S GENETIC SPORADIC 5365 03:53:05,320 --> 03:53:05,920 CLINICAL TRIALS AND RESEARCH 5366 03:53:05,920 --> 03:53:08,360 NOW. 5367 03:53:08,360 --> 03:53:10,400 ONE OF THE GOALS WE PUT TOGETHER 5368 03:53:10,400 --> 03:53:13,000 WAS THE IMPORTANCE OF LEARNING 5369 03:53:13,000 --> 03:53:14,440 FROM EVERYBODY WITH ILLNESS 5370 03:53:14,440 --> 03:53:15,440 WHETHER IT'S SOMEBODY WHO 5371 03:53:15,440 --> 03:53:25,600 CARRIES THE GENE, AT RISK OR AND 5372 03:53:25,600 --> 03:53:27,880 ESPECIALLY IN THE RARE DISEASE 5373 03:53:27,880 --> 03:53:31,520 AND WE CAN SPEED THE PROGRESS. 5374 03:53:31,520 --> 03:53:34,000 WE KNOW FROM OTHER FIELDS THAT 5375 03:53:34,000 --> 03:53:37,200 WHEN A FIELD DEVELOPS 5376 03:53:37,200 --> 03:53:38,520 UNDERSTANDING WITH CLINICAL 5377 03:53:38,520 --> 03:53:39,520 TOOLS TO UNDERSTAND DIFFERENT 5378 03:53:39,520 --> 03:53:41,840 BIOLOGY IN PEOPLE OR TO DEVELOP 5379 03:53:41,840 --> 03:53:43,200 BETTER OUTCOMES MEASURES TO 5380 03:53:43,200 --> 03:53:45,040 FOLLOW THE ILLNESS AND TELL US 5381 03:53:45,040 --> 03:53:46,240 IF THE DRUG WORKS OR BETTER 5382 03:53:46,240 --> 03:53:49,040 FLUID OR DIGITAL BIOMARKERS TO 5383 03:53:49,040 --> 03:53:51,240 SUBSET PEOPLE THE THERAPY 5384 03:53:51,240 --> 03:53:55,240 DEVELOPMENT JUST GETS SPED UP 5385 03:53:55,240 --> 03:53:55,760 TREMENDOUSLY. 5386 03:53:55,760 --> 03:53:57,480 IN PARTICULAR DEVELOPING THINGS 5387 03:53:57,480 --> 03:53:59,280 LIKE SURROGATE MARKERS WHERE IF 5388 03:53:59,280 --> 03:54:01,800 YOUR DRUG CAN CHANGE A MARKER IN 5389 03:54:01,800 --> 03:54:03,520 THREE MONTHS AND PREDICT 5390 03:54:03,520 --> 03:54:06,440 LONG-TERM OR CLINICAL AFFECTS 5391 03:54:06,440 --> 03:54:09,160 YOU CAN SEE THE DRUG DEVELOPMENT 5392 03:54:09,160 --> 03:54:09,640 TREMENDOUSLY. 5393 03:54:09,640 --> 03:54:11,560 WE HAVE AN ILLNESS VERY HETRO 5394 03:54:11,560 --> 03:54:13,560 GENEIC BETWEEN PEOPLE AND WE 5395 03:54:13,560 --> 03:54:16,360 DON'T HAVE SHORT TERM MARKERS TO 5396 03:54:16,360 --> 03:54:16,960 PREDICT LONG-TERM CLINICAL 5397 03:54:16,960 --> 03:54:19,440 AFFECT AND WE DON'T KNOW HOW TO 5398 03:54:19,440 --> 03:54:20,320 INTERVENE EARLY YET. 5399 03:54:20,320 --> 03:54:22,320 SO OUR GROUP REALLY FOCUSSED ON 5400 03:54:22,320 --> 03:54:25,440 WHAT COULD WE DO TO SOLVE THE 5401 03:54:25,440 --> 03:54:27,400 FUNDAMENTAL QUESTIONS. 5402 03:54:27,400 --> 03:54:30,840 IT CAME DOWN TO THE IMPORTANCE 5403 03:54:30,840 --> 03:54:33,400 OF WELL-DONE NATURAL HISTORY 5404 03:54:33,400 --> 03:54:33,640 STUDIES. 5405 03:54:33,640 --> 03:54:35,480 YOU CAN LEARN A LOT FROM A 5406 03:54:35,480 --> 03:54:38,200 NATURAL HISTORY STUDY AND WE 5407 03:54:38,200 --> 03:54:40,680 WERE BLESSED TO HAVE 5408 03:54:40,680 --> 03:54:42,480 EPIDEMIOLOGISTS ON THE COMMITTEE 5409 03:54:42,480 --> 03:54:45,080 TO GIVE US INPUT ON THAT. 5410 03:54:45,080 --> 03:54:49,000 WHAT WE CAN LEARN FROM A NATURAL 5411 03:54:49,000 --> 03:54:50,360 HISTORY STUDY ARE SEVERAL FOLD 5412 03:54:50,360 --> 03:54:51,320 AND WELL GO THROUGH THESE IN 5413 03:54:51,320 --> 03:54:52,560 MORE DETAILS. 5414 03:54:52,560 --> 03:54:55,000 ONE IS UNDERSTANDING THE 5415 03:54:55,000 --> 03:54:56,640 HETEROGENEITY BETWEEN PEOPLE AND 5416 03:54:56,640 --> 03:54:58,760 UNDERSTANDING THE COURSE OF THE 5417 03:54:58,760 --> 03:55:01,600 ILLNESS THAT CAN PROVIDE DATA TO 5418 03:55:01,600 --> 03:55:04,320 PEOPLE LIKE DR. QUINTANA AND 5419 03:55:04,320 --> 03:55:06,680 ANOTHER STATISTICIANS TO COME UP 5420 03:55:06,680 --> 03:55:09,760 WITH TO DO EARLY TRIAL DESIGN. 5421 03:55:09,760 --> 03:55:15,400 WE CAN DEVELOP BETTER WAYS TO 5422 03:55:15,400 --> 03:55:17,320 ASSESS WHETHER A DRUG WORKS OR 5423 03:55:17,320 --> 03:55:21,760 NOT AND LOOKING WHETHER EARLY 5424 03:55:21,760 --> 03:55:24,680 SCIENCE CAN INTERVENE EARLY. 5425 03:55:24,680 --> 03:55:26,240 THOSE ARE SOME THINGS WE CAN 5426 03:55:26,240 --> 03:55:28,320 LEARN AND KEY PRINCIPLES THAT 5427 03:55:28,320 --> 03:55:30,240 CAN DEVELOP IN THE FIELD OF 5428 03:55:30,240 --> 03:55:37,800 ROBUST NATURAL HISTORY STUDY 5429 03:55:37,800 --> 03:55:44,200 APPROACH. 5430 03:55:44,200 --> 03:55:46,680 AND WE KNOW THERE ARE NATURAL 5431 03:55:46,680 --> 03:55:47,720 HISTORIES GOING ON FOR PEOPLE AT 5432 03:55:47,720 --> 03:55:49,760 RISK FOR THE ILLNESS BUT NOWHERE 5433 03:55:49,760 --> 03:55:55,240 NEAR THE SIZE AND NEED TO HAVE A 5434 03:55:55,240 --> 03:55:57,040 HUGE IMPACT. 5435 03:55:57,040 --> 03:55:57,760 THERE ARE NATURAL HISTORY 5436 03:55:57,760 --> 03:56:04,080 STUDIES FUNDED BY THE FDA IN 5437 03:56:04,080 --> 03:56:07,160 PEOPLE WITH SYMPTOMATIC STUDIES 5438 03:56:07,160 --> 03:56:10,200 AND HAVE TO BE LARGER FOR IMPACT 5439 03:56:10,200 --> 03:56:12,440 AND GET DATA REFLECTED THROUGH 5440 03:56:12,440 --> 03:56:17,640 MEDICAL RECORDS OR INSURANCE 5441 03:56:17,640 --> 03:56:17,920 COMPANIES. 5442 03:56:17,920 --> 03:56:28,440 THERE'S CREATIVE WAYS TO ANSWER 5443 03:56:35,680 --> 03:56:39,880 KEY QUESTIONS AND GAPS IN 5444 03:56:39,880 --> 03:56:40,160 KNOWLEDGE. 5445 03:56:40,160 --> 03:56:42,800 SO OUR PRIMARY AND WAS THE 5446 03:56:42,800 --> 03:56:45,400 ESTABLISHMENT OF A ROBUST 5447 03:56:45,400 --> 03:56:46,680 NATURAL HISTORY STUDY THAT WOULD 5448 03:56:46,680 --> 03:56:52,720 ENROLL PEOPLE AT RISK FOR THE 5449 03:56:52,720 --> 03:56:57,760 ILLNESS, PEOPLE PRE-SYMPTOMATIC 5450 03:56:57,760 --> 03:57:01,640 AND ASYMPTOMATIC AND PROVIDE 5451 03:57:01,640 --> 03:57:04,640 INFORMATION CRITICAL FOR 5452 03:57:04,640 --> 03:57:07,720 SPEEDING UP THERAPY DEVELOPMENT 5453 03:57:07,720 --> 03:57:08,920 FOR ALS AND WITH THAT I'LL PASS 5454 03:57:08,920 --> 03:57:11,520 IT TO TIM TO TALK ABOUT PRIORITY 5455 03:57:11,520 --> 03:57:13,840 2. 5456 03:57:13,840 --> 03:57:16,320 >>PRIORITIES 2 AND 3 DERIVED 5457 03:57:16,320 --> 03:57:17,640 FROM THE LARGE NATURAL HISTORY 5458 03:57:17,640 --> 03:57:19,640 STUDY AND THE GOAL ESTABLISHING 5459 03:57:19,640 --> 03:57:21,000 THAT TO THEN ENABLE SOME OF THE 5460 03:57:21,000 --> 03:57:23,840 OTHER THINGS. 5461 03:57:23,840 --> 03:57:26,200 SO ENABLE BETTER OUTCOME 5462 03:57:26,200 --> 03:57:28,320 MEASURES AND BIOMARKERS IN 5463 03:57:28,320 --> 03:57:29,600 INDIVIDUALS WITH DISEASE AND 5464 03:57:29,600 --> 03:57:31,120 THOSE AT RISK. 5465 03:57:31,120 --> 03:57:33,760 TO HAVE MORE DETAIL BEHIND THAT, 5466 03:57:33,760 --> 03:57:37,360 I THINK DEVELOPING BETTER TRIAL 5467 03:57:37,360 --> 03:57:39,480 METHODOLOGY AND ONE OF THE KEY 5468 03:57:39,480 --> 03:57:41,600 IDEAS IS THE MOST EFFICIENT USE 5469 03:57:41,600 --> 03:57:43,520 OF RESOURCES FOR CLINICAL TRIALS 5470 03:57:43,520 --> 03:57:44,480 RESPECTING THE PARTICIPANT'S 5471 03:57:44,480 --> 03:57:45,920 TIME AND ENERGY THAT GOES INTO 5472 03:57:45,920 --> 03:57:47,600 THAT AND OTHER RESOURCES THAT GO 5473 03:57:47,600 --> 03:57:51,320 INTO THAT AS WELL. 5474 03:57:51,320 --> 03:57:51,960 THINKING ABOUT NOVEL CLINICAL 5475 03:57:51,960 --> 03:57:52,600 TRIAL DESIGNS. 5476 03:57:52,600 --> 03:57:56,920 ONE WOULD BE THE PLATFORM TRIAL. 5477 03:57:56,920 --> 03:58:00,600 I THINK YOU ALL KNOW OTHERS HAVE 5478 03:58:00,600 --> 03:58:02,480 PIONEERED AND THERE MAY BE OTHER 5479 03:58:02,480 --> 03:58:06,120 TRIAL DESIGNS AND WAYS TO RUN 5480 03:58:06,120 --> 03:58:06,600 TRIALS. 5481 03:58:06,600 --> 03:58:07,920 I THINK ALSO NOVEL WAYS TO SHARE 5482 03:58:07,920 --> 03:58:10,440 AND ENHANCE THE GATHERING OF 5483 03:58:10,440 --> 03:58:10,640 DATA. 5484 03:58:10,640 --> 03:58:13,800 WAYS WE CAN SHARE DATA FOR 5485 03:58:13,800 --> 03:58:17,040 PLACEBO TREATMENTS AND WAYS TO 5486 03:58:17,040 --> 03:58:17,600 ENHANCE CLINICAL TRIAL BY 5487 03:58:17,600 --> 03:58:19,640 SHARING DATA AND I THINK ALSO 5488 03:58:19,640 --> 03:58:21,000 WITHIN THAT MORE BROADLY 5489 03:58:21,000 --> 03:58:21,880 INCLUSIVE THERE WAS A QUESTION 5490 03:58:21,880 --> 03:58:27,560 THAT CAME IN ABOUT ENROLLING 5491 03:58:27,560 --> 03:58:29,960 PEOPLE IN RURAL AREAS. 5492 03:58:29,960 --> 03:58:31,760 OUR AIM IS TO BE MORE INCLUSIVE 5493 03:58:31,760 --> 03:58:32,320 OF MORE GROUPS IN CLINICAL 5494 03:58:32,320 --> 03:58:34,320 TRIALS. 5495 03:58:34,320 --> 03:58:36,960 THE OTHER IS DEVELOPING OUTCOME 5496 03:58:36,960 --> 03:58:37,760 MEASURES. 5497 03:58:37,760 --> 03:58:39,640 I THINK WE ALL RECOGNIZE THAT 5498 03:58:39,640 --> 03:58:42,560 THERE IS ROOM FOR IMPROVEMENT OF 5499 03:58:42,560 --> 03:58:45,400 A FUNCTIONAL OUTCOME MEASURE. 5500 03:58:45,400 --> 03:58:46,800 I THINK WE ALSO ALL RECOGNIZE 5501 03:58:46,800 --> 03:58:48,320 THAT WE NEED A FUNCTIONAL 5502 03:58:48,320 --> 03:58:49,800 OUTCOME MEASURE. 5503 03:58:49,800 --> 03:58:53,040 SO WE HAVE ONE, THE ALS SCALE 5504 03:58:53,040 --> 03:58:55,800 AND CAN THAT BE IMPROVED OR DO 5505 03:58:55,800 --> 03:58:57,000 WE NEED TO DEVELOP SOMETHING 5506 03:58:57,000 --> 03:58:57,200 ELSE. 5507 03:58:57,200 --> 03:58:58,800 SO FUNCTIONAL OUTCOME MEASURE 5508 03:58:58,800 --> 03:59:01,120 AND NOVEL OUTCOME MEASURES AND 5509 03:59:01,120 --> 03:59:03,080 THERE'S A WHOLE VARIETY OF THESE 5510 03:59:03,080 --> 03:59:05,720 PROPOSED THAT WE CAN USE AND 5511 03:59:05,720 --> 03:59:07,400 SOME BEING EXPLORE AND HAVEN'T 5512 03:59:07,400 --> 03:59:08,520 IN THE SETTING OF ALS AND MAYBE 5513 03:59:08,520 --> 03:59:10,800 WE CAN BRING THESE IN FROM OTHER 5514 03:59:10,800 --> 03:59:18,120 FIELDS WHETHER IT'S FROM CANCER 5515 03:59:18,120 --> 03:59:19,000 OR OTHER NEURODEGENERATIVE 5516 03:59:19,000 --> 03:59:20,280 DISEASES AND ONE IS DIGITAL 5517 03:59:20,280 --> 03:59:23,400 HEALTH AND THOSE ARE JUST NOW 5518 03:59:23,400 --> 03:59:24,920 BEING TAPPED INTO THAT MAY 5519 03:59:24,920 --> 03:59:26,560 INCLUDE SOME AT-HOME MEASURES AS 5520 03:59:26,560 --> 03:59:27,440 WELL. 5521 03:59:27,440 --> 03:59:28,920 AGAIN, NOVEL MEASURES. 5522 03:59:28,920 --> 03:59:30,160 AND BIOMARKERS. 5523 03:59:30,160 --> 03:59:31,800 THERE'S BEEN A LOT OF DISCUSSION 5524 03:59:31,800 --> 03:59:33,560 ABOUT BIOMARKERS ALREADY. 5525 03:59:33,560 --> 03:59:34,520 RECOGNIZING THE POWER OF THIS 5526 03:59:34,520 --> 03:59:37,760 LARGE STUDY TO HELP DEVELOP 5527 03:59:37,760 --> 03:59:38,840 MARKERS. 5528 03:59:38,840 --> 03:59:40,960 ALSO WANT TO THINK ABOUT 5529 03:59:40,960 --> 03:59:44,520 PHARMACO DYNAMIC STUDIES SO WE 5530 03:59:44,520 --> 03:59:47,600 KNOW WHETHER THE DRUG HIT ITS 5531 03:59:47,600 --> 03:59:50,160 TARGET AND MAY MEAN BROAD 5532 03:59:50,160 --> 03:59:52,760 DEVELOPMENT ACROSS STUDIES. 5533 03:59:52,760 --> 03:59:53,840 AND ONE WE'RE MOST INTERESTED IN 5534 03:59:53,840 --> 03:59:56,400 IS MARKERS OF SUCCESS AT THE 5535 03:59:56,400 --> 03:59:57,600 EARLIEST TIME POINT. 5536 03:59:57,600 --> 04:00:03,280 IN MY OWN OPINION I THINK IF 5537 04:00:03,280 --> 04:00:05,280 NEURAL FILAMENT IS A GOOD SIGN 5538 04:00:05,280 --> 04:00:08,000 AND AN EARLY MARKER OF SUCCESS 5539 04:00:08,000 --> 04:00:15,680 BUT IS THAT THE ONLY MARKER AND 5540 04:00:15,680 --> 04:00:17,240 HAU COULD WE DEVELOP MARKERS 5541 04:00:17,240 --> 04:00:20,040 THAT WOULD BE ABLE TO SEE EACH 5542 04:00:20,040 --> 04:00:22,680 OF THE DIFFERENT AREAS THAT 5543 04:00:22,680 --> 04:00:25,000 MIGHT BE TREATED BY A DRUG THAT 5544 04:00:25,000 --> 04:00:27,240 WAS HELPFUL FOR ALS. 5545 04:00:27,240 --> 04:00:32,000 AND THEN LASTLY WE'VE INCLUDED 5546 04:00:32,000 --> 04:00:32,840 THIS DISEASE THOSE WITH DISEASE 5547 04:00:32,840 --> 04:00:33,640 AND AT RISK. 5548 04:00:33,640 --> 04:00:36,440 THIS IS A MAJOR PART OF OUR 5549 04:00:36,440 --> 04:00:37,400 THINKING. 5550 04:00:37,400 --> 04:00:39,840 H 5551 04:00:39,840 --> 04:00:41,880 HERE WE NEED TO INCLUDE THOSE AT 5552 04:00:41,880 --> 04:00:43,400 RISK AND THIS HAS COME UP 5553 04:00:43,400 --> 04:00:45,280 SEVERAL TIMES TODAY BUT 5554 04:00:45,280 --> 04:00:46,360 INCLUDING THOSE AT RISK AND 5555 04:00:46,360 --> 04:00:50,920 THERE ARE A NUMBER OF SMALL 5556 04:00:50,920 --> 04:00:53,240 STUDIES AND NEIL SCHNEIDER 5557 04:00:53,240 --> 04:00:55,040 HIGHLIGHTED THOSE AND WE NEED TO 5558 04:00:55,040 --> 04:00:57,760 EXPAND THOSE AND GROW THOSE AND 5559 04:00:57,760 --> 04:01:04,000 MAKE THOSE MORE PROMINENT. 5560 04:01:04,000 --> 04:01:14,560 PRIORITY NUMBER 3, TO FACILITATE 5561 04:01:17,680 --> 04:01:28,160 EARLIER INVENTION AND IMPROVE 5562 04:01:28,960 --> 04:01:32,240 DIAGNOSIS AND DISCOVER THE 5563 04:01:32,240 --> 04:01:33,440 EARLIEST MANIFESTATIONS OF ALS 5564 04:01:33,440 --> 04:01:40,000 AND THE NON-GENETIC ALS CAN WE 5565 04:01:40,000 --> 04:01:41,640 DIAGNOSE THAT SOONER AND THERE'S 5566 04:01:41,640 --> 04:01:43,160 WAYS PEOPLE WHO THOUGHT ABOUT 5567 04:01:43,160 --> 04:01:45,760 HOW TO MAKE THE DIAGNOSES 5568 04:01:45,760 --> 04:01:47,960 SOONER, ONE IS GETTING SOMEONE 5569 04:01:47,960 --> 04:01:49,760 TO AN ALS CENTER AND GETTING 5570 04:01:49,760 --> 04:01:53,640 SOMEONE TO A GROUP THAT'S GOING 5571 04:01:53,640 --> 04:02:01,280 TO RECOGNIZE THE DISEASE. 5572 04:02:01,280 --> 04:02:03,160 THAT WOULD BE HELPFUL IN 5573 04:02:03,160 --> 04:02:04,080 UNDERSTANDING WHETHER IT'S 5574 04:02:04,080 --> 04:02:06,240 ASSOCIATED WITH ALS OR THE 5575 04:02:06,240 --> 04:02:07,600 MECHANICS OF REFERRALS OR 5576 04:02:07,600 --> 04:02:09,800 GETTING PEOPLE TO THE RIGHT 5577 04:02:09,800 --> 04:02:10,000 PLACE. 5578 04:02:10,000 --> 04:02:12,640 AND THEN EARLIER INVENTION. 5579 04:02:12,640 --> 04:02:14,120 I THINK IT'S OBVIOUS WHEN STATED 5580 04:02:14,120 --> 04:02:17,960 THAT MOST OF US WOULD AGREE -- 5581 04:02:17,960 --> 04:02:21,760 MAYBE NOT EVERYONE BUT I THINK 5582 04:02:21,760 --> 04:02:25,760 ALL OF US WOULD AGREE IF SOMEONE 5583 04:02:25,760 --> 04:02:34,360 IS ASYMPTOMATIC STARTING SOMEONE 5584 04:02:34,360 --> 04:02:41,600 EARLIER AND GETTING DIAGNOSIS 5585 04:02:41,600 --> 04:02:44,360 EARLIER AND SOONER IS ALMOST 5586 04:02:44,360 --> 04:02:47,640 ALWAYS BETTER AND STUDYING THE 5587 04:02:47,640 --> 04:02:48,440 EARLIEST MANIFESTATIONS FROM ALS 5588 04:02:48,440 --> 04:02:51,880 AND WE WANT TO STUDY EARLY ALS 5589 04:02:51,880 --> 04:02:53,920 OR THE BEGINS BOTH IN THE 5590 04:02:53,920 --> 04:02:56,080 NON-GENETIC FORM AND IN THE 5591 04:02:56,080 --> 04:02:56,880 GENETIC FORM. 5592 04:02:56,880 --> 04:02:59,960 BUT WHEN WE LOOK AT SOMEONE WHO 5593 04:02:59,960 --> 04:03:02,080 IS SYMPTOMATIC WITH ALS THE 5594 04:03:02,080 --> 04:03:03,720 WORLD OF MOTOR NEURONS IS 5595 04:03:03,720 --> 04:03:05,600 ALREADY ON FIRE. 5596 04:03:05,600 --> 04:03:09,560 COOPER PENNER SAID EXPANDING 5597 04:03:09,560 --> 04:03:11,840 EXPONEN 5598 04:03:11,840 --> 04:03:12,200 EXPONENTIALLY. 5599 04:03:12,200 --> 04:03:18,880 THE OPPORTUNITY BY MOVING THIS 5600 04:03:18,880 --> 04:03:20,040 AS EARLY AS POSSIBLE TO SEE WHAT 5601 04:03:20,040 --> 04:03:22,680 STARTED THE FIRE AND MAYBE WE 5602 04:03:22,680 --> 04:03:25,320 CAN PUT OUT THE FIRE AND DISEASE 5603 04:03:25,320 --> 04:03:29,760 BEFORE IT GROWS INTO THE AWFUL 5604 04:03:29,760 --> 04:03:31,600 DISEASE THAT IS SYMPTOMATIC ALS. 5605 04:03:31,600 --> 04:03:33,800 AND WITH THAT I'M GOING TURN IT 5606 04:03:33,800 --> 04:03:39,280 BACK TO MERIT. 5607 04:03:39,280 --> 04:03:39,760 >>THANK YOU. 5608 04:03:39,760 --> 04:03:41,200 OUR GROUP ALSO DISCUSSED WAYS TO 5609 04:03:41,200 --> 04:03:44,640 KEEP LEARNING ABOUT THE ILLNESS 5610 04:03:44,640 --> 04:03:46,400 AND ACCELERATE HOW WE GO THROUGH 5611 04:03:46,400 --> 04:03:48,280 WHAT NOW A RICH PIPELINE OF 5612 04:03:48,280 --> 04:03:50,720 THERAPEUTIC IDEAS IN THE FIELD. 5613 04:03:50,720 --> 04:03:56,520 AND I KNOW MANY KNOW ABOUT THE 5614 04:03:56,520 --> 04:03:59,640 PLATFORM TRIALS AND THIS IS ONE 5615 04:03:59,640 --> 04:04:01,800 WAY OF BEING MORE EFFICIENT IN 5616 04:04:01,800 --> 04:04:04,080 HOW WE TEST TREATMENTS IN PEOPLE 5617 04:04:04,080 --> 04:04:05,760 WITH ALS PROVIDING MORE ACCESS 5618 04:04:05,760 --> 04:04:09,600 TO PEOPLE AND CONTINUING TO 5619 04:04:09,600 --> 04:04:13,120 LEARN ABOUT THE ILLNESS THROUGH 5620 04:04:13,120 --> 04:04:13,760 CLINICAL TRIALS AND THIS 5621 04:04:13,760 --> 04:04:14,360 COMPLEMENTS NATURAL HISTORY 5622 04:04:14,360 --> 04:04:24,520 STUDIES. 5623 04:04:36,680 --> 04:04:38,920 AND YOU SHARE DATA AND NO MATTER 5624 04:04:38,920 --> 04:04:41,880 THE OUTCOME OF THE MEASURES AND 5625 04:04:41,880 --> 04:04:43,120 YOU'RE LEARNING ABOUT THE 5626 04:04:43,120 --> 04:04:44,320 ILLNESS AND BIOLOGY AND 5627 04:04:44,320 --> 04:04:46,720 DEVELOPING NEW BIOMARKERS. 5628 04:04:46,720 --> 04:04:47,280 WE KNOW FOR EXAMPLE FROM 5629 04:04:47,280 --> 04:04:49,400 PLATFORM TRIALS AND ONCOLOGY 5630 04:04:49,400 --> 04:04:51,480 THAT NOT ONLY DID THEY GET DRUGS 5631 04:04:51,480 --> 04:04:53,600 TO MARKET THROUGH THAT WAY AND A 5632 04:04:53,600 --> 04:04:56,560 MUCH FASTER WAY AND LOWER COST 5633 04:04:56,560 --> 04:04:59,160 BUT ALSO DEVELOPED THOSE 5634 04:04:59,160 --> 04:05:00,080 SURROGATE BIOMARKERS. 5635 04:05:00,080 --> 04:05:01,800 SO THE FIRST STUDIES MAY HAVE 5636 04:05:01,800 --> 04:05:07,240 BEEN LONGER BUT DEVELOPED 5637 04:05:07,240 --> 04:05:09,200 SHORT-TERM MEASURES OF SUCCESS 5638 04:05:09,200 --> 04:05:10,280 AND EXCITING ADVANCE IN ALS IS 5639 04:05:10,280 --> 04:05:12,520 THERE'S NOW THREE DIFFERENT 5640 04:05:12,520 --> 04:05:14,520 APPROACHES OF THIS GOING ON 5641 04:05:14,520 --> 04:05:16,560 THROUGHOUT THE WORLD. 5642 04:05:16,560 --> 04:05:18,680 AND THE GROUPS DOING THESE TYPES 5643 04:05:18,680 --> 04:05:20,160 OF INNOVATIVE TRIALS ARE TALKING 5644 04:05:20,160 --> 04:05:22,760 TO EACH OTHER AND SHARING AND 5645 04:05:22,760 --> 04:05:24,120 LEARNING BEST PRACTICES TO 5646 04:05:24,120 --> 04:05:25,160 CONTINUE TO TEST THE MOST 5647 04:05:25,160 --> 04:05:26,560 EXCITING THINGS THAT COME FROM 5648 04:05:26,560 --> 04:05:29,640 BASIC SCIENCE LABS AND 5649 04:05:29,640 --> 04:05:32,600 TRANSLATION GROUPS. 5650 04:05:32,600 --> 04:05:35,640 WITH THAT WE'D LIKE TO SPEND OUR 5651 04:05:35,640 --> 04:05:37,240 REMAINING TIME ANSWERING AS MANY 5652 04:05:37,240 --> 04:05:38,480 QUESTIONS AS POSSIBLE ABOUT THE 5653 04:05:38,480 --> 04:05:40,080 PRIORITIES IDENTIFIED BY OUR 5654 04:05:40,080 --> 04:05:40,840 WORKING GROUP. 5655 04:05:40,840 --> 04:05:42,600 PLEASE FEEL FREE TO CONTINUE TO 5656 04:05:42,600 --> 04:05:45,160 ENTER QUESTIONS USING THE ZOOM 5657 04:05:45,160 --> 04:05:47,720 Q&A BOX OR IN THE QUESTIONS AND 5658 04:05:47,720 --> 04:05:48,800 COMMENTS FORM AVAILABLE ON THE 5659 04:05:48,800 --> 04:05:49,640 MEETING WEBSITE. 5660 04:05:49,640 --> 04:05:51,120 I HOPE WE'LL HAVE TIME TO ANSWER 5661 04:05:51,120 --> 04:05:54,840 ALL THE QUESTIONS. 5662 04:05:54,840 --> 04:05:57,360 BUT WE'RE GOING PLEASE KNOW 5663 04:05:57,360 --> 04:05:58,640 WE'RE COLLECTING ALL QUESTIONS 5664 04:05:58,640 --> 04:06:03,800 TO IN OUR WORK GROG FORWARD. 5665 04:06:03,800 --> 04:06:04,800 -- GOING FORWARD. 5666 04:06:04,800 --> 04:06:06,640 WE COMBINED QUESTION ARE SIMILAR 5667 04:06:06,640 --> 04:06:08,560 BUT AGAIN WILL TRY TO ANSWER ALL 5668 04:06:08,560 --> 04:06:09,200 OF THEM. 5669 04:06:09,200 --> 04:06:16,160 >>AND WITH THAT BEING SAID 5670 04:06:16,160 --> 04:06:17,400 THANK YOU, I'M GOING JUMP TO THE 5671 04:06:17,400 --> 04:06:20,120 FIRST QUESTION SUBMITTED IN 5672 04:06:20,120 --> 04:06:24,720 ADVANCE AND IT'S FOR JEREMY. 5673 04:06:24,720 --> 04:06:27,040 JEREMY, WHAT IS YOUR 5674 04:06:27,040 --> 04:06:32,040 RECOMMENDATION OR YOUR PLAN, 5675 04:06:32,040 --> 04:06:33,920 TIMETABLE FOR A ROBUST ALS 5676 04:06:33,920 --> 04:06:44,280 NATURAL HISTORY STUDY? 5677 04:06:45,600 --> 04:06:49,480 I DON'T KNOW I HAVE A SPECIFIC 5678 04:06:49,480 --> 04:06:51,320 TIMETABLE AND ONE HAS BEEN 5679 04:06:51,320 --> 04:06:53,240 APPROACHED BY THE ALS COMMUNITY 5680 04:06:53,240 --> 04:06:58,560 ALREADY LOOKING AT GENE CARRIERS 5681 04:06:58,560 --> 04:07:09,040 AND FOLLOWING PEOPLE THEY'RE 5682 04:07:09,480 --> 04:07:11,040 SYMPTOMATIC WITH ALS. 5683 04:07:11,040 --> 04:07:13,200 THOSE STUDIES EXIST. 5684 04:07:13,200 --> 04:07:15,480 BECAUSE OF THE RARITY OF EACH 5685 04:07:15,480 --> 04:07:17,600 INDIVIDUAL GENETIC CAUSE OF ALS 5686 04:07:17,600 --> 04:07:20,040 WITH THE POTENTIAL EXCEPTION OF 5687 04:07:20,040 --> 04:07:27,200 C9, THEY HAVE TO BE VERY 5688 04:07:27,200 --> 04:07:28,440 BROAD-BASED BECAUSE THERE'S NOT 5689 04:07:28,440 --> 04:07:31,120 HAVE MANY PATIENTS TO ACCUMULATE 5690 04:07:31,120 --> 04:07:32,080 TO THOSE STUDIES. 5691 04:07:32,080 --> 04:07:34,120 IN THE QUESTIONS THAT CAN BE 5692 04:07:34,120 --> 04:07:41,640 ASKED OF THE STUDIES IS WELL, 5693 04:07:41,640 --> 04:07:45,600 WHEN ONE IS PRE-SYMPTOMATIC HOW 5694 04:07:45,600 --> 04:07:46,760 INTENSE SHOULD THE EVALUATION BE 5695 04:07:46,760 --> 04:07:47,960 AND WHAT SHOULD WE KNOW ABOUT 5696 04:07:47,960 --> 04:07:51,040 SYMPTOMS THAT OCCUR THAT GIVE US 5697 04:07:51,040 --> 04:07:52,640 THE INFORMATION ABOUT WHEN THE 5698 04:07:52,640 --> 04:07:54,880 FIRE'S ABOUT TO START? 5699 04:07:54,880 --> 04:07:56,120 BUT I THINK THE TIMETABLE FOR 5700 04:07:56,120 --> 04:08:05,800 THAT IS NOW. 5701 04:08:05,800 --> 04:08:07,800 THERE'S NEEDS TO BE 5702 04:08:07,800 --> 04:08:09,240 AMPLIFICATION IN TERMS OF SIZE 5703 04:08:09,240 --> 04:08:12,160 AND IN TERMS OF THE 5704 04:08:12,160 --> 04:08:13,080 INTENSIVENESS WITH WHICH 5705 04:08:13,080 --> 04:08:17,640 PARTICIPANTS ARE EVALUATED PRIOR 5706 04:08:17,640 --> 04:08:18,760 TO THIS. 5707 04:08:18,760 --> 04:08:26,480 THAT ONLY COVERS 10% OR MORE OF 5708 04:08:26,480 --> 04:08:29,640 PEOPLE WITH ALS AND IS IT MORE 5709 04:08:29,640 --> 04:08:31,080 COMPLICATED FOR THOSE WHOM WE 5710 04:08:31,080 --> 04:08:35,120 CAN'T IDENTIFY EASILY IN A 5711 04:08:35,120 --> 04:08:38,320 PRE-SYMPTOMATIC WAY. 5712 04:08:38,320 --> 04:08:40,400 THERE'S ONLY TWO OPTIONS I 5713 04:08:40,400 --> 04:08:42,360 PERCEIVE AND WOULD BE GREAT TO 5714 04:08:42,360 --> 04:08:47,840 UNDERSTAND MORE AND ONE IS TO 5715 04:08:47,840 --> 04:08:50,240 LOOK AT A GENERAL POPULATION 5716 04:08:50,240 --> 04:08:53,240 THIS IS DONE IN COHORT STUDIES 5717 04:08:53,240 --> 04:08:56,040 IN GENEALOGY AND FOR ALS WOULD 5718 04:08:56,040 --> 04:08:56,960 HAVE TO BE LARGE. 5719 04:08:56,960 --> 04:08:59,680 IF YOU THINK OF INSTITUTES A 5720 04:08:59,680 --> 04:09:04,880 COHORT TO LOOK PRIMARILY AT 5721 04:09:04,880 --> 04:09:06,920 EARLY ONSET ALS WHAT'S THE 5722 04:09:06,920 --> 04:09:08,440 SAMPLE SIZE? 5723 04:09:08,440 --> 04:09:11,000 YOU THINK PERHAPS IF YOU ENROLL 5724 04:09:11,000 --> 04:09:12,760 A MILLION PEOPLE ABOUT 60 MAY 5725 04:09:12,760 --> 04:09:15,600 HAVE ALS AND MAYBE THE SAME 5726 04:09:15,600 --> 04:09:16,520 AMOUNT MAY BE PRE-SYMPTOMATIC. 5727 04:09:16,520 --> 04:09:17,640 THAT MAY BE ENOUGH. 5728 04:09:17,640 --> 04:09:20,200 YOU MAY NEED A COUPLE MILLION OR 5729 04:09:20,200 --> 04:09:20,760 10 MILLION. 5730 04:09:20,760 --> 04:09:23,120 IN ORDER TO DO THAT I THINK THE 5731 04:09:23,120 --> 04:09:27,040 ALS COMMUNITY HAS TO PARTNER 5732 04:09:27,040 --> 04:09:30,240 WITH OTHER GROUPS INTERESTED IN 5733 04:09:30,240 --> 04:09:33,760 PRE-SYMPTOMATIC ONSET OF 5734 04:09:33,760 --> 04:09:34,080 DISEASE. 5735 04:09:34,080 --> 04:09:35,840 MAYBE A NEURODEGENERATIVE 5736 04:09:35,840 --> 04:09:36,920 DISEASE RESEARCH CONSORTIUM TO 5737 04:09:36,920 --> 04:09:41,680 TRY TO WORK HARD TO ISOLATE AND 5738 04:09:41,680 --> 04:09:46,240 IDENTIFY PRE-SYMPTOMATIC, EARLY 5739 04:09:46,240 --> 04:09:47,640 SYMPTOMATIC FOLKS IN A VARIETY 5740 04:09:47,640 --> 04:09:49,760 OF DISEASES TO COMMAND THE SIZE 5741 04:09:49,760 --> 04:09:51,520 OF THE CONSORTIUM AND HAVE TO 5742 04:09:51,520 --> 04:09:53,640 DECIDE WHAT'S A MINIMALLY 5743 04:09:53,640 --> 04:09:55,400 CRITICAL SET OF EVALUATIONS TO 5744 04:09:55,400 --> 04:09:56,240 APPLY TO THESE PEOPLE OVER TIME 5745 04:09:56,240 --> 04:09:56,840 RECOGNIZING IT'S MILLIONS OF 5746 04:09:56,840 --> 04:10:07,000 PEOPLE. 5747 04:10:09,880 --> 04:10:11,680 AND I KNOW THERE'S MORE EXPERT 5748 04:10:11,680 --> 04:10:13,760 IN DESIGN BUT IT'S A DIRECTION 5749 04:10:13,760 --> 04:10:24,000 WE HAVE TO GO. 5750 04:10:32,440 --> 04:10:39,080 AND GO BACK AND IDENTIFY THROUGH 5751 04:10:39,080 --> 04:10:48,000 BLOOD SAMPLES AND GO BACK TO SEE 5752 04:10:48,000 --> 04:10:49,520 WHAT LIFE WAS LIKE PRIOR TO 5753 04:10:49,520 --> 04:10:49,800 SYMPTOMS. 5754 04:10:49,800 --> 04:10:53,680 THAT'S A MORE LIMITED APPROACH 5755 04:10:53,680 --> 04:10:55,760 BUT MAY BE MORE FEASIBLE. 5756 04:10:55,760 --> 04:10:58,520 THOSE ARE THE OPTIONS I SEE. 5757 04:10:58,520 --> 04:11:02,560 >>MAY I MAKE A QUICK FOLLOW-UP 5758 04:11:02,560 --> 04:11:03,160 BREAKING ALL THE RULES RIGHT 5759 04:11:03,160 --> 04:11:11,640 NOW. 5760 04:11:11,640 --> 04:11:16,960 CAN NEURODE GENE RATION BORROW 5761 04:11:16,960 --> 04:11:20,040 THE -- NEURODEGENERATION BORROW 5762 04:11:20,040 --> 04:11:22,080 THE MODEL FROM OTHER DISEASES 5763 04:11:22,080 --> 04:11:32,600 AND DOES THAT EXIST ANYWHERE? 5764 04:11:38,200 --> 04:11:39,680 >>IF YOU LOOK AT PAST LARGE 5765 04:11:39,680 --> 04:11:44,920 COHORTS THEY WEREN'T DESIGNED TO 5766 04:11:44,920 --> 04:11:48,280 LOOK AT SPECIFIC DISEASE STATES. 5767 04:11:48,280 --> 04:11:49,440 THERE'S THE NURSES HEALTH STUDY 5768 04:11:49,440 --> 04:11:53,680 WHERE A LARGE GROUP OF PEOPLE 5769 04:11:53,680 --> 04:12:03,400 VOLUNTEERED TO BE PERIODICALLY 5770 04:12:03,400 --> 04:12:05,440 FOLLOWED AND WE'VE HAD SUCCESSES 5771 04:12:05,440 --> 04:12:07,600 WITH RESPECT TO ALS WITH COHORTS 5772 04:12:07,600 --> 04:12:12,640 AND THERE WAS A LARGE 5773 04:12:12,640 --> 04:12:14,040 CARDIOVASCULAR COHORT THAT 5774 04:12:14,040 --> 04:12:19,160 IDENTIFIED PEOPLE WITH THE EARLY 5775 04:12:19,160 --> 04:12:23,400 1800s AND PEOPLE WITH 5776 04:12:23,400 --> 04:12:24,680 PARTICIPATION DURING THAT TIME 5777 04:12:24,680 --> 04:12:26,320 OF SERVICE HAD AN INCREASED RISK 5778 04:12:26,320 --> 04:12:32,760 OF ALS AND IT WAS IN A LARGE 5779 04:12:32,760 --> 04:12:35,720 MULTIPLE COHORT PRIMARILY 5780 04:12:35,720 --> 04:12:36,600 DIRECTED TOWARDS CARDIOVASCULAR 5781 04:12:36,600 --> 04:12:37,800 DISEASE AND OTHER DISEASE 5782 04:12:37,800 --> 04:12:40,840 ENCOUNTERS AS WELL. 5783 04:12:40,840 --> 04:12:43,520 THAT'S THE BEST I CAN DO. 5784 04:12:43,520 --> 04:12:49,520 >>MY NEXT QUESTION IS FOR TIM. 5785 04:12:49,520 --> 04:12:51,160 WILL ALS RESEARCH BE OPTIMIZED 5786 04:12:51,160 --> 04:12:54,520 TO LOOK AT MULTIPLE CURES IN 5787 04:12:54,520 --> 04:12:57,760 PARALLEL RATHER THAN A SLOW AND 5788 04:12:57,760 --> 04:13:08,120 SEQUENTIAL APPROACH? 5789 04:13:17,680 --> 04:13:19,440 >>THERE ARE MULTIPLE TRIALS AND 5790 04:13:19,440 --> 04:13:22,880 MULTIPLE COMPANIES RUNNING 5791 04:13:22,880 --> 04:13:24,560 TRIALS AND AND A BUNCH MORE 5792 04:13:24,560 --> 04:13:26,720 TRIALS COMING THROUGH AND I WANT 5793 04:13:26,720 --> 04:13:27,360 TO ACKNOWLEDGE THE PLATFORM 5794 04:13:27,360 --> 04:13:33,760 TRIAL MANY OF YOU KNOW ABOUT AND 5795 04:13:33,760 --> 04:13:35,240 LAUNCH A PLATFORM TRIAL AND I 5796 04:13:35,240 --> 04:13:37,480 THINK IT'S AC SELT RATING OUR 5797 04:13:37,480 --> 04:13:38,320 ABILITY TO GET DRUGS IN THE 5798 04:13:38,320 --> 04:13:38,720 TRIAL. 5799 04:13:38,720 --> 04:13:41,640 MAYBE YOU CAN SPEAK TO THAT MORE 5800 04:13:41,640 --> 04:13:44,560 THAN I WOULD. 5801 04:13:44,560 --> 04:13:45,800 WHETHER IT REACHED THE 5802 04:13:45,800 --> 04:13:46,600 ACCELERATION WE THOUGHT IT WOULD 5803 04:13:46,600 --> 04:13:49,000 TO GET MORE DRUGS IN AND TEST 5804 04:13:49,000 --> 04:13:50,000 MORE DRUGS. 5805 04:13:50,000 --> 04:13:51,160 I THINK THEY'RE HAPPENING 5806 04:13:51,160 --> 04:13:51,400 QUICKLY. 5807 04:13:51,400 --> 04:13:55,400 I THINK THEY'RE HAPPENING IN 5808 04:13:55,400 --> 04:13:57,760 PARALLEL RATHER THAN MULTIPLE 5809 04:13:57,760 --> 04:14:07,560 EFFORTS GOING ON ALL AT ONCE. 5810 04:14:07,560 --> 04:14:09,760 >>THE NEXT QUESTION IS ALSO FOR 5811 04:14:09,760 --> 04:14:11,680 TIM, THERE'S A FEW DIFFERENT 5812 04:14:11,680 --> 04:14:17,640 PARTS TIM, MULTIPLE PEOPLE 5813 04:14:17,640 --> 04:14:18,760 SUBMITTED QUESTIONS ABOUT 5814 04:14:18,760 --> 04:14:23,280 CLINICAL TRIALS FOR PREVENTING 5815 04:14:23,280 --> 04:14:26,040 ALS IN PEOPLE WHO HAVE GENE 5816 04:14:26,040 --> 04:14:31,240 MUTATIONS BUT DON'T YET HAVE 5817 04:14:31,240 --> 04:14:31,520 SYMPTOMS. 5818 04:14:31,520 --> 04:14:33,480 SO FIRST PART OF THIS, WHY ARE 5819 04:14:33,480 --> 04:14:37,800 THERE NO TRIALS TESTING WHETHER 5820 04:14:37,800 --> 04:14:40,240 CURRENT ALS THERAPIES PREVENT 5821 04:14:40,240 --> 04:14:45,200 DISEASE IN PEOPLE WHO WERE AT 5822 04:14:45,200 --> 04:14:53,640 HIGH RISK OF DEVELOPING ALS? 5823 04:14:53,640 --> 04:15:03,880 >> 5824 04:15:03,880 --> 04:15:06,200 >>AND I PUT IN THE LINK TO THE 5825 04:15:06,200 --> 04:15:06,880 ACCESS TRIAL THAT DESCRIBES THE 5826 04:15:06,880 --> 04:15:11,680 TRIAL IN MORE DETAIL. 5827 04:15:11,680 --> 04:15:17,520 THERE'S A TRIAL BY THE ACADEMIC 5828 04:15:17,520 --> 04:15:19,160 INVESTIGATOR LOOKING AT PEOPLE 5829 04:15:19,160 --> 04:15:21,760 WHO HAVE MUTATIONS AND LOOKING 5830 04:15:21,760 --> 04:15:24,320 WHETHER THEY HAVE A BIOMARKERS 5831 04:15:24,320 --> 04:15:32,240 RISE IN FILAMENT TO GET FIRST ON 5832 04:15:32,240 --> 04:15:33,800 TO A DRUG TO FOCUS ON PROTEIN 5833 04:15:33,800 --> 04:15:35,520 AND THERE'S A NEW TRIAL FOR US 5834 04:15:35,520 --> 04:15:36,200 IN THE ALS WORLD. 5835 04:15:36,200 --> 04:15:40,880 THERE MAY BE OTHERS COMING 5836 04:15:40,880 --> 04:15:44,280 THROUGH AS WE DEVELOP OTHER 5837 04:15:44,280 --> 04:15:49,760 GENETIC FOCUSSED THERAPIES. 5838 04:15:49,760 --> 04:15:50,920 THERE'S AN INTERESTING TRIAL 5839 04:15:50,920 --> 04:15:52,520 DESIGN AND CHALLENGING TO RUN 5840 04:15:52,520 --> 04:15:53,680 WE'LL GET TO IN A SECOND, I 5841 04:15:53,680 --> 04:15:56,560 THINK. 5842 04:15:56,560 --> 04:16:02,360 >>WHAT ARE THE CHALLENGES FOR 5843 04:16:02,360 --> 04:16:12,880 CONDUCTING PREVENTION TRIALS? 5844 04:16:21,840 --> 04:16:24,200 >>IN SOME DISEASE WHEN YOU GET 5845 04:16:24,200 --> 04:16:25,400 SICK SEEMS TO BE CLOSELY LINKED 5846 04:16:25,400 --> 04:16:26,960 TO WHEN OTHER PEOPLE IN YOUR 5847 04:16:26,960 --> 04:16:28,640 FAMILY GET SICK. 5848 04:16:28,640 --> 04:16:29,560 FOR EXAMPLE, PLUS OR MINUS FIVE 5849 04:16:29,560 --> 04:16:31,000 YEARS AND YOU CAN USE THAT 5850 04:16:31,000 --> 04:16:33,440 INFORMATION TO MAKE PREDICTIONS 5851 04:16:33,440 --> 04:16:34,960 AND MAYBE GET SOMEBODY ON A 5852 04:16:34,960 --> 04:16:36,800 THERAPY CLOSE TO THE TIME WHEN 5853 04:16:36,800 --> 04:16:39,040 YOUR FAMILY TENDS TO GET 5854 04:16:39,040 --> 04:16:39,280 DISEASE. 5855 04:16:39,280 --> 04:16:41,240 IN ALS THERE'S A WIDE RANGE. 5856 04:16:41,240 --> 04:16:43,080 IT COULD BE 40 YEARS DIFFERENCE 5857 04:16:43,080 --> 04:16:43,960 AND DOESN'T MAKE SENSE TO 5858 04:16:43,960 --> 04:16:50,280 INTEREST PEOPLE ON A DRUG FOR 40 5859 04:16:50,280 --> 04:16:50,800 YEARS. 5860 04:16:50,800 --> 04:16:52,040 WHAT WAS BROUGHT UP BEFORE IS 5861 04:16:52,040 --> 04:16:53,560 WHEN IS SOMEONE CONVERTING OR 5862 04:16:53,560 --> 04:16:55,920 HOW DO WE KNOW SOMEONE'S ABOUT 5863 04:16:55,920 --> 04:17:00,760 TO CONVERT TO SYMPTOMATIC ALS? 5864 04:17:00,760 --> 04:17:07,560 AND NEURO FILAMENT MAY BE THE 5865 04:17:07,560 --> 04:17:08,080 MARKER. 5866 04:17:08,080 --> 04:17:09,280 IT'S NOT CLEAR FOR OTHER FORMS 5867 04:17:09,280 --> 04:17:11,200 OF DISEASE AND MAYBE WE NEED 5868 04:17:11,200 --> 04:17:13,800 OTHER MARKERS COMING THROUGH AND 5869 04:17:13,800 --> 04:17:17,760 IT WILL COME FROM THE NATURAL 5870 04:17:17,760 --> 04:17:19,000 HISTORIES STUDIES AND UNDERSTAND 5871 04:17:19,000 --> 04:17:24,400 POPULATION AND TESTING MULTIPLE 5872 04:17:24,400 --> 04:17:24,640 MARKERS. 5873 04:17:24,640 --> 04:17:25,760 THE MAIN CHALLENGE IS HOW DO WE 5874 04:17:25,760 --> 04:17:28,320 KNOW THEY'RE ABOUT TO CONVERT 5875 04:17:28,320 --> 04:17:30,720 BECAUSE IT DOESN'T MAKE SENSE TO 5876 04:17:30,720 --> 04:17:32,000 KEEP THEM ON DRUG FOR LONG 5877 04:17:32,000 --> 04:17:37,760 PERIOD OF TIME, DECADES. 5878 04:17:37,760 --> 04:17:48,320 I THINK YOU TOUCHED ON THIS IN 5879 04:17:59,320 --> 04:18:00,520 THE STUDIES. 5880 04:18:00,520 --> 04:18:04,120 >>STEP 1 IS GENETIC 5881 04:18:04,120 --> 04:18:04,960 COUNSELLING. 5882 04:18:04,960 --> 04:18:07,000 TO PEOPLE AT RISK FOR CARRYING A 5883 04:18:07,000 --> 04:18:10,320 GENE BUT DON'T YET KNOW WHETHER 5884 04:18:10,320 --> 04:18:12,600 THEY HAVE A GENE SHOULD GO 5885 04:18:12,600 --> 04:18:14,840 THROUGH A FORMALIZED FOR 5886 04:18:14,840 --> 04:18:15,440 CAREFUL, THOUGHTFUL PROCESS 5887 04:18:15,440 --> 04:18:19,280 ABOUT IDENTIFYING WHETHER OR NOT 5888 04:18:19,280 --> 04:18:22,040 THEY CARRY THE GENE. 5889 04:18:22,040 --> 04:18:23,440 THAT COULD BE DONE CLINICALLY 5890 04:18:23,440 --> 04:18:25,040 AND DONE IN RESEARCH STUDIES AND 5891 04:18:25,040 --> 04:18:26,520 THE OTHER WAY TO GET PEOPLE IN 5892 04:18:26,520 --> 04:18:35,680 IS A NUMBER OF RESEARCH STUDIES 5893 04:18:35,680 --> 04:18:39,520 AND UNIVERSITY OF MIAMI AND 5894 04:18:39,520 --> 04:18:40,120 WASH-U WITH JAMES BERRY AND 5895 04:18:40,120 --> 04:18:50,240 MYSELF. 5896 04:18:52,440 --> 04:18:53,640 AND I'D ANYTHING THE GENE 5897 04:18:53,640 --> 04:18:54,560 THROUGH GENETIC STUDIES OR 5898 04:18:54,560 --> 04:19:00,200 COUNSEL SELL -- COUNSELLING. 5899 04:19:00,200 --> 04:19:02,120 >>HOW CAN MORE BE DONE TO FIND 5900 04:19:02,120 --> 04:19:05,680 OUT AT WHAT STAGE AND HOW EARLY 5901 04:19:05,680 --> 04:19:06,760 MODIFYING TREATMENTS SHOULD 5902 04:19:06,760 --> 04:19:09,760 BEGIN? 5903 04:19:09,760 --> 04:19:11,280 >>GOOD QUESTION. 5904 04:19:11,280 --> 04:19:13,120 RIGHT NOW WE'RE TREATING PEOPLE 5905 04:19:13,120 --> 04:19:18,240 WHEN THEY HAVE CLINICALLY 5906 04:19:18,240 --> 04:19:21,800 OBVIOUS ALS BUT AS EARLY AS 5907 04:19:21,800 --> 04:19:24,600 POSSIBLE UNDERSTANDING A LOT ARE 5908 04:19:24,600 --> 04:19:26,280 AIMED AT SLOWING PROGRESSION AND 5909 04:19:26,280 --> 04:19:27,480 IT'S BEEN TOUCHED ON WE THINK IF 5910 04:19:27,480 --> 04:19:29,680 WE'RE ABLE TO IDENTIFY PEOPLE 5911 04:19:29,680 --> 04:19:31,760 EARLIER THERE COULD BE A BENEFIT 5912 04:19:31,760 --> 04:19:33,440 TO TREATING EARLIER. 5913 04:19:33,440 --> 04:19:35,640 A LOT OF WHAT'S BEEN DISCUSSED 5914 04:19:35,640 --> 04:19:37,600 IDENTIFYING EIGHT RISK PEOPLE 5915 04:19:37,600 --> 04:19:41,840 AND EXPEDITING DIAGNOSIS AND 5916 04:19:41,840 --> 04:19:44,760 TIME TO REFERRAL TO A TERTIARY 5917 04:19:44,760 --> 04:19:46,560 CENTER ARE CRITICAL TO FIND 5918 04:19:46,560 --> 04:19:48,360 PEOPLE EARLIER WHERE THEY MIGHT 5919 04:19:48,360 --> 04:19:49,560 BENEFIT MORE FROM SOME OF THE 5920 04:19:49,560 --> 04:19:57,600 TREATMENTS BEING TESTED. 5921 04:19:57,600 --> 04:19:59,680 ALSO WE KNOW THEY'RE GOOD AT 5922 04:19:59,680 --> 04:20:00,560 MEASURING CHANGE IN THE MIDDLE 5923 04:20:00,560 --> 04:20:03,240 RANGE AND DEVELOPING THINGS MORE 5924 04:20:03,240 --> 04:20:07,320 RESPONSIVE TO CHANGE EARLY IN 5925 04:20:07,320 --> 04:20:09,760 DISEASE AND EVEN BEFORE WE'RE 5926 04:20:09,760 --> 04:20:11,280 CURRENTLY DIAGNOSING PATIENTS IS 5927 04:20:11,280 --> 04:20:14,960 CRITICAL AND WE HEARD FROM 5928 04:20:14,960 --> 04:20:16,920 PATIENT ADVOCATES PATIENTS 5929 04:20:16,920 --> 04:20:19,160 EXCLUDED INTEREST TRIALS AND 5930 04:20:19,160 --> 04:20:20,960 CONSIDERED ADVANCED STAGES WANT 5931 04:20:20,960 --> 04:20:22,960 ACCESS TO TREATMENT TOO AND IS A 5932 04:20:22,960 --> 04:20:24,800 GROUP TO CONSIDER IN THIS AND 5933 04:20:24,800 --> 04:20:32,520 NOT LEAVE OUT IN RESEARCH 5934 04:20:32,520 --> 04:20:32,760 EFFORTS. 5935 04:20:32,760 --> 04:20:35,760 I DON'T THINK THERE'S AN ANSWER 5936 04:20:35,760 --> 04:20:36,720 AND MAY BE DRUG SPECIFIC. 5937 04:20:36,720 --> 04:20:40,880 >>DO YOU THINK WE NEED IT 5938 04:20:40,880 --> 04:20:43,120 CHANGE OUR DIAGNOSTIC CRITERIA 5939 04:20:43,120 --> 04:20:45,280 AND THE REASON I ASK IS I AM 5940 04:20:45,280 --> 04:20:47,720 SEEING PEOPLE EARLIER IN THE 5941 04:20:47,720 --> 04:20:48,160 ILLNESS. 5942 04:20:48,160 --> 04:20:50,520 THEY DON'T QUITE MEET THE 5943 04:20:50,520 --> 04:20:51,680 CRITERIA. 5944 04:20:51,680 --> 04:20:53,560 I'M WONDERING WHETHER WE COULD 5945 04:20:53,560 --> 04:20:57,080 WORK ON REVISED CRITERIA FOR 5946 04:20:57,080 --> 04:21:00,200 EARLIER DIAGNOSIS. 5947 04:21:00,200 --> 04:21:02,400 >>I SHARE THE CONCERN ABOUT 5948 04:21:02,400 --> 04:21:05,680 USING THE SCORE OF CRITERIA TO 5949 04:21:05,680 --> 04:21:07,280 MAKE THE DIAGNOSIS. 5950 04:21:07,280 --> 04:21:11,000 IT DOES INCLUDE EXCLUDE PEOPLE 5951 04:21:11,000 --> 04:21:13,000 WITH EARLY ALS WHICH DON'T HAVE 5952 04:21:13,000 --> 04:21:20,320 ENOUGH REGIONAL SPREAD BUT HAVE 5953 04:21:20,320 --> 04:21:20,520 ALS. 5954 04:21:20,520 --> 04:21:23,240 WE'D LIKE A TEST TO DETERMINE 5955 04:21:23,240 --> 04:21:27,160 ALS BUT WE STILL RELY ON CLINIC 5956 04:21:27,160 --> 04:21:29,680 EXAM AND IT DOES MAKE THINGS 5957 04:21:29,680 --> 04:21:30,560 TRICKY. 5958 04:21:30,560 --> 04:21:31,760 IN THE FUTURE HOPEFULLY WE'LL 5959 04:21:31,760 --> 04:21:35,400 HAVE MORE OBJECTIVE MARKERS BUT 5960 04:21:35,400 --> 04:21:37,600 RELYING ON THIS CRITERIA ALONE 5961 04:21:37,600 --> 04:21:40,080 IS NOT WISE BECAUSE WE'LL BE 5962 04:21:40,080 --> 04:21:42,240 EXCLUDING PATIENTS WHO CLEARLY 5963 04:21:42,240 --> 04:21:44,960 HAVE ALS BUT DON'T HAVE ENOUGH 5964 04:21:44,960 --> 04:21:45,680 REGIONAL SPREAD YET AND I DON'T 5965 04:21:45,680 --> 04:21:47,680 THINK THERE'S A BENEFIT TO THAT. 5966 04:21:47,680 --> 04:21:51,760 >>THANK YOU. 5967 04:21:51,760 --> 04:21:55,600 THE NEXT QUESTION IS FOR MERIT. 5968 04:21:55,600 --> 04:21:58,520 WHAT IS THE STATUS OF RESEARCH 5969 04:21:58,520 --> 04:22:05,000 AND TRIALS ON THE C9 MUTATION. 5970 04:22:05,000 --> 04:22:07,600 >>I'M VERY EXCITED ABOUT THE 5971 04:22:07,600 --> 04:22:10,360 RESEARCH GOING ON WITH C9. 5972 04:22:10,360 --> 04:22:13,040 MANY THINGS FROM AT-RISK AND 5973 04:22:13,040 --> 04:22:14,160 NATURAL HISTORY STUDIES AND 5974 04:22:14,160 --> 04:22:15,680 THERAPEUTIC STUDIES. 5975 04:22:15,680 --> 04:22:17,840 ALMOST CALLING OUT FOR OUR 5976 04:22:17,840 --> 04:22:19,680 PLATFORM C9. 5977 04:22:19,680 --> 04:22:21,760 I'M NOT VOLUNTEERING TO PUT THAT 5978 04:22:21,760 --> 04:22:23,400 TOGETHER BUT OFFERING TO HELP 5979 04:22:23,400 --> 04:22:23,640 OTHERS. 5980 04:22:23,640 --> 04:22:25,680 THERE'S A COUPLE GENE THERAPY 5981 04:22:25,680 --> 04:22:30,440 TRIALS IN C9. 5982 04:22:30,440 --> 04:22:32,960 WE DID KNOW THE BIO GEN ONE HAS 5983 04:22:32,960 --> 04:22:35,960 STOPPED AND SOME ARE BEING 5984 04:22:35,960 --> 04:22:37,760 ENROLLED IN THE TRIAL AND 5985 04:22:37,760 --> 04:22:40,560 THERE'S SMALL MOLECULE STUDIES 5986 04:22:40,560 --> 04:22:44,120 GOING ON IN C9 AND ONE IS ON 5987 04:22:44,120 --> 04:22:44,720 A.I. THERAPEUTICS. 5988 04:22:44,720 --> 04:22:48,800 THOSE ARE ALL ACTIVELY ENROLLING 5989 04:22:48,800 --> 04:22:50,520 THERAPEUTICS BUT MANY ARE 5990 04:22:50,520 --> 04:22:54,160 WORKING ON C9 SPECIFIC 5991 04:22:54,160 --> 04:22:54,480 TREATMENTS. 5992 04:22:54,480 --> 04:22:57,040 I THINK IT'S IMPORTANT FOR 5993 04:22:57,040 --> 04:22:58,560 PEOPLE TO KNOW AND WITH THE 5994 04:22:58,560 --> 04:23:02,720 COUNSELLING IF THEY CARRY THE 5995 04:23:02,720 --> 04:23:04,440 GENE AND FIND OUT ABOUT THE 5996 04:23:04,440 --> 04:23:05,520 TRIALS AND FOR THERE TO BE AN 5997 04:23:05,520 --> 04:23:08,280 EASY WAY TO LEARN ABOUT STUDIES. 5998 04:23:08,280 --> 04:23:10,160 AND ONE OF THE CHALLENGES WE'RE 5999 04:23:10,160 --> 04:23:11,640 FINDING IS WE'RE STARTING TO 6000 04:23:11,640 --> 04:23:16,720 DOUGH -- DO THE TRIALS OF C9 IS 6001 04:23:16,720 --> 04:23:18,840 WE DON'T HAVE ALL THE OUTCOME 6002 04:23:18,840 --> 04:23:20,600 MEASURES TO CAPTURE ALL THE 6003 04:23:20,600 --> 04:23:21,760 COMPLEXITY OF C9. 6004 04:23:21,760 --> 04:23:26,000 IT'S BEEN BRINGING TOGETHER THE 6005 04:23:26,000 --> 04:23:30,360 WORLDS OF ALS AND DEVELOPING 6006 04:23:30,360 --> 04:23:31,800 WAYS FOR COMMUNITIES TO COME 6007 04:23:31,800 --> 04:23:33,600 TOGETHER AND MANY TRIALS OF 6008 04:23:33,600 --> 04:23:36,360 ENROLLING THOSE WITH C9 AND ALS 6009 04:23:36,360 --> 04:23:39,360 AND THOSE WHO HAVE FRONTAL 6010 04:23:39,360 --> 04:23:41,920 DISRUPTION OR BOTH WE NEED TO 6011 04:23:41,920 --> 04:23:45,760 TELL SUCCESS UPON ALL THE 6012 04:23:45,760 --> 04:23:49,760 POPULATIONS. 6013 04:23:49,760 --> 04:23:57,760 >>THE NEXT QUESTION IS FOR BOB. 6014 04:23:57,760 --> 04:23:59,120 ARE THERE COLLABORATIONS FOR 6015 04:23:59,120 --> 04:24:03,040 CLINICAL RESEARCH TO FIND A CURE 6016 04:24:03,040 --> 04:24:04,640 AND IF THERE ARE, ARE THERE WAYS 6017 04:24:04,640 --> 04:24:09,760 TO IMPROVE THE COLLABORATION 6018 04:24:09,760 --> 04:24:11,880 BETWEEN COUNTRIES? 6019 04:24:11,880 --> 04:24:14,720 >>I THINK WE'RE DOING A GOOD 6020 04:24:14,720 --> 04:24:17,120 JOB BUT SO MUCH MORE WE CAN DO 6021 04:24:17,120 --> 04:24:17,680 BETTER. 6022 04:24:17,680 --> 04:24:22,680 WITH COLLABORATIONS ACROSS THE 6023 04:24:22,680 --> 04:24:26,840 BOUNDARIES OF BORDERS AND 6024 04:24:26,840 --> 04:24:27,720 BETWEEN NATIONS THERE'S A NUMBER 6025 04:24:27,720 --> 04:24:31,440 OF STUDIES ONGOING AND FUNDED 6026 04:24:31,440 --> 04:24:35,680 WITHIN THE UNITED STATES OR FROM 6027 04:24:35,680 --> 04:24:39,040 OTHER COUNTRIES THAT INVOLVE 6028 04:24:39,040 --> 04:24:40,160 U.S. INVESTIGATORS AND CLINICAL 6029 04:24:40,160 --> 04:24:41,400 CLINICIANS AND RESEARCHERS AND 6030 04:24:41,400 --> 04:24:43,560 THERE'S A LARGE NUMBER, TOO MANY 6031 04:24:43,560 --> 04:24:44,720 TO TRY TO LIST. 6032 04:24:44,720 --> 04:24:49,280 GIVEN THAT, THERE'S MORE THAT 6033 04:24:49,280 --> 04:24:51,280 CAN BE DONE WITH THE SENSE OF 6034 04:24:51,280 --> 04:24:52,280 SHARING DATA AND INFORMATION AND 6035 04:24:52,280 --> 04:24:54,080 SHARING RESOURCES AND TOOLS THAT 6036 04:24:54,080 --> 04:25:04,200 CAN GO ACROSS THE BORDERS. 6037 04:25:04,200 --> 04:25:09,080 WE'RE GETTING BETTER AND BETTER 6038 04:25:09,080 --> 04:25:11,680 AT INFORMATION AND RESOURCES AND 6039 04:25:11,680 --> 04:25:18,560 THERE'S EXAMPLES ON THE RESOURCE 6040 04:25:18,560 --> 04:25:21,120 SIDE AND THAT'S BIO FLUIDS AND 6041 04:25:21,120 --> 04:25:24,120 SAMPLES SHARED DURING CLINICAL 6042 04:25:24,120 --> 04:25:30,200 TRIALS AND THE ANSWER CONSORTIUM 6043 04:25:30,200 --> 04:25:36,240 SHARING LINES FROM -- AND 6044 04:25:36,240 --> 04:25:37,360 TARGETED ALS ANOTHER NATURAL 6045 04:25:37,360 --> 04:25:43,280 HISTORIES STUDY AND SHARING THE 6046 04:25:43,280 --> 04:25:49,040 SAMPLES AND SHARING POST MORTEM 6047 04:25:49,040 --> 04:25:49,680 INFORMATION AND ONE CHALLENGE 6048 04:25:49,680 --> 04:25:51,800 THAT REMAINS AS WE GENERATE MORE 6049 04:25:51,800 --> 04:25:54,640 AND MORE INFORMATION AROUND THE 6050 04:25:54,640 --> 04:25:59,360 DATA IS HOWE TO SHARE THE DATA 6051 04:25:59,360 --> 04:25:59,600 RESULTS. 6052 04:25:59,600 --> 04:26:02,200 THAT TAKES EFFORTS ONGOING FROM 6053 04:26:02,200 --> 04:26:05,440 A LARGE NUMBER OF INDIVIDUALS TO 6054 04:26:05,440 --> 04:26:08,080 PUT THEM ON PLATFORM THAT'S 6055 04:26:08,080 --> 04:26:09,280 SHARABLE BUT THE CHALLENGE 6056 04:26:09,280 --> 04:26:11,520 BECOMES THAT IF WE LOOK AT 6057 04:26:11,520 --> 04:26:12,160 DIFFERENT MEASURES IF PEOPLE 6058 04:26:12,160 --> 04:26:13,760 AREN'T DOING THEM IN A 6059 04:26:13,760 --> 04:26:17,360 STANDARDIZED APPROACH AND METHOD 6060 04:26:17,360 --> 04:26:19,080 IT'S HARD TO SHARE THE 6061 04:26:19,080 --> 04:26:19,560 INFORMATION. 6062 04:26:19,560 --> 04:26:21,360 IT'S CREATING STANDARD OPERATING 6063 04:26:21,360 --> 04:26:23,200 PROCEDURES AND KEEPING THEM 6064 04:26:23,200 --> 04:26:24,400 INCORPORATED INTO OUR RESEARCH 6065 04:26:24,400 --> 04:26:27,280 STUDIES THAT WILL ENABLE FURTHER 6066 04:26:27,280 --> 04:26:29,280 EVOLUTION OF THE TYPES OF DATA 6067 04:26:29,280 --> 04:26:34,880 THAT IS SHARED AND THE RESOURCES 6068 04:26:34,880 --> 04:26:38,200 THAT ARE GENERATED AND THE DOWN 6069 04:26:38,200 --> 04:26:41,840 STREAM ABILITY WITH THE CLINICAL 6070 04:26:41,840 --> 04:26:43,240 COURSE OF DISEASE PROGRESSION. 6071 04:26:43,240 --> 04:26:47,200 WE'RE DOING A LOT BUT I THINK 6072 04:26:47,200 --> 04:26:49,120 THERE ARE AVENUES WE CAN ENHANCE 6073 04:26:49,120 --> 04:26:51,120 TO BETTER FACILITATE THE SHARING 6074 04:26:51,120 --> 04:27:01,320 AND INTERNATIONAL COLLABORATION. 6075 04:27:01,320 --> 04:27:05,680 >>I FOUND IT COOL EARLIER WHEN 6076 04:27:05,680 --> 04:27:09,080 WALTER WAS TALKING ABOUT CREATE 6077 04:27:09,080 --> 04:27:17,680 CONSORTIUM IS LOOKING AT DIVERSE 6078 04:27:17,680 --> 04:27:18,320 POPULATION AND GOING TO SOUTH 6079 04:27:18,320 --> 04:27:21,760 AFRICA AND CREATE IS LOOKING AT 6080 04:27:21,760 --> 04:27:24,480 THE GENETIC UNDER PINNINGS OF 6081 04:27:24,480 --> 04:27:26,080 THOSE WITH ALS. 6082 04:27:26,080 --> 04:27:28,040 THAT WAS NEAT TO HEAR. 6083 04:27:28,040 --> 04:27:30,280 >>I'M GOING BREAK RULES AND 6084 04:27:30,280 --> 04:27:34,800 RESPOND TO WHAT YOU JUST SAID. 6085 04:27:34,800 --> 04:27:37,080 I THINK AS WE GO FORWARD THERE 6086 04:27:37,080 --> 04:27:39,520 ARE WAYS AND MEANS AND GROUPS 6087 04:27:39,520 --> 04:27:41,480 THAT ARE VERY INTERESTED IN EVEN 6088 04:27:41,480 --> 04:27:44,280 WITH OUR ONGOING AND UPCOMING 6089 04:27:44,280 --> 04:27:45,360 NATURAL HISTORY STUDIES TO BE 6090 04:27:45,360 --> 04:27:46,840 MORE INCLUSIVE AND DIFFERENT 6091 04:27:46,840 --> 04:27:49,760 WAYS TO REACH OUT WITHIN BOTH 6092 04:27:49,760 --> 04:27:50,800 THE DIVERSE POPULATIONS BOTH 6093 04:27:50,800 --> 04:27:54,560 WITHIN THIS COUNTRY AND AS YOU 6094 04:27:54,560 --> 04:27:55,840 JUST MENTIONED INCLUDING OTHER 6095 04:27:55,840 --> 04:27:56,880 NATIONS. 6096 04:27:56,880 --> 04:27:57,920 OTHER COUNTRIES, OTHER REGIONS 6097 04:27:57,920 --> 04:28:00,320 OF THE WORLD INTO THE TYPES OF 6098 04:28:00,320 --> 04:28:02,600 STUDIES SO WE GAIN GREATER 6099 04:28:02,600 --> 04:28:05,680 INSIDE INTO THE DIVERSITY OF ALS 6100 04:28:05,680 --> 04:28:16,160 ACROSS PATIENT POPULATIONS. 6101 04:28:20,760 --> 04:28:22,400 >>THIS IS FOR TIM AND MERIT. 6102 04:28:22,400 --> 04:28:23,680 WHAT IS THE DIFFERENCE BETWEEN A 6103 04:28:23,680 --> 04:28:27,280 THERAPY AND A CURE? 6104 04:28:27,280 --> 04:28:29,680 >>WELL, IT'S INTERESTING IN THE 6105 04:28:29,680 --> 04:28:30,880 SECOND PART OF THAT IS HELPING 6106 04:28:30,880 --> 04:28:33,760 THINK ABOUT CURE. 6107 04:28:33,760 --> 04:28:37,120 I LOVE THIS QUESTION. 6108 04:28:37,120 --> 04:28:38,600 IT'S A GREAT REMINDER TO ALL OF 6109 04:28:38,600 --> 04:28:40,480 US TO GO FOR CURES AND THAT'S 6110 04:28:40,480 --> 04:28:42,080 WHY WE'RE WORKING ON IT AND WE 6111 04:28:42,080 --> 04:28:43,400 TALK TO COLLEAGUES AROUND THE 6112 04:28:43,400 --> 04:28:44,520 NATION THAT WORK ON ALS, THAT'S 6113 04:28:44,520 --> 04:28:45,600 WHAT THEY'RE THINKING ABOUT, 6114 04:28:45,600 --> 04:28:47,680 THEY'RE THINKING ABOUT CURES. 6115 04:28:47,680 --> 04:28:48,720 THE DIFFERENCE BETWEEN A 6116 04:28:48,720 --> 04:28:50,320 TREATMENT AND CURE IS A 6117 04:28:50,320 --> 04:28:51,960 TREATMENT IS PARTIALLY SLOWING 6118 04:28:51,960 --> 04:28:54,520 THINGS DOWN AND A CURE STOPS THE 6119 04:28:54,520 --> 04:28:55,840 DISEASE IN ITS TRACKS. 6120 04:28:55,840 --> 04:29:01,000 I'LL COMMENT IN A NUMBER OF 6121 04:29:01,000 --> 04:29:01,760 OTHER DISORDERS THERE'S 6122 04:29:01,760 --> 04:29:03,280 TREATMENT ONE WAS OKAY, 6123 04:29:03,280 --> 04:29:04,920 TREATMENT TWO ADDED IN AND MADE 6124 04:29:04,920 --> 04:29:06,560 IT BETTER AND TREATMENT ONCE YOU 6125 04:29:06,560 --> 04:29:08,360 GET THREE AND SOMETIMES FOUR 6126 04:29:08,360 --> 04:29:10,280 SOMETIMES IT TAKES MORE THAN ONE 6127 04:29:10,280 --> 04:29:12,520 TREATMENT AND IF YOU GET TO 6128 04:29:12,520 --> 04:29:14,240 THREE, FOUR, REALLY GOOD 6129 04:29:14,240 --> 04:29:15,880 TREATMENTS, YOU MIGHT END UP 6130 04:29:15,880 --> 04:29:16,640 WITH A CURE. 6131 04:29:16,640 --> 04:29:19,760 AND SO I THINK THAT WE'RE MOVING 6132 04:29:19,760 --> 04:29:20,520 THAT WAY. 6133 04:29:20,520 --> 04:29:22,080 AND YOU WILL OF US ARE REALISTIC 6134 04:29:22,080 --> 04:29:27,600 ABOUT HOW MUCH CAN WE MOVE THE 6135 04:29:27,600 --> 04:29:29,360 BAR IN THE CLINICAL RESEARCH AND 6136 04:29:29,360 --> 04:29:31,080 IN THIS TOUGH DISEASE THAT'S 6137 04:29:31,080 --> 04:29:33,120 BEEN RESISTANT TO A HOST OF 6138 04:29:33,120 --> 04:29:36,200 CLINICAL TRIALS. 6139 04:29:36,200 --> 04:29:40,040 I THINK WE NEED TO BE REALISTIC 6140 04:29:40,040 --> 04:29:41,880 BOTH AIM FOR CURES, ACCEPT 6141 04:29:41,880 --> 04:29:43,840 TREATMENTS AND HOPEFULLY PUT THE 6142 04:29:43,840 --> 04:29:44,520 TREATMENTS TOGETHER THAT WILL 6143 04:29:44,520 --> 04:29:47,680 END UP AS A SECURE. 6144 04:29:47,680 --> 04:29:49,760 I'LL PASS IT TO MERIT. 6145 04:29:49,760 --> 04:29:52,520 >>COMPLETELY. 6146 04:29:52,520 --> 04:29:56,200 WE'RE LOOKING TO FIND TREATMENT 6147 04:29:56,200 --> 04:29:59,080 THAT WILL STOP PROGRESSION AND 6148 04:29:59,080 --> 04:29:59,920 BRING BACK FUNCTION AND TO STOP 6149 04:29:59,920 --> 04:30:01,440 THEM FROM STARTING. 6150 04:30:01,440 --> 04:30:03,400 ALL IS THIS COMPLEX AND WE'LL 6151 04:30:03,400 --> 04:30:04,840 PROBABLY FOR NOW TAKE MANY 6152 04:30:04,840 --> 04:30:05,680 THERAPIES TO GET US THERE BUT 6153 04:30:05,680 --> 04:30:09,480 THERE COULD BE A DAY WHERE WE 6154 04:30:09,480 --> 04:30:10,600 UNDERSTAND ENOUGH ABOUT THE 6155 04:30:10,600 --> 04:30:11,960 SCIENCE WHERE IT WILL BE 6156 04:30:11,960 --> 04:30:12,560 SIMPLER. 6157 04:30:12,560 --> 04:30:15,320 AGAIN, CANCER IS A GREAT MODEL 6158 04:30:15,320 --> 04:30:17,240 WHERE FOR SOME CANCERS ARE 6159 04:30:17,240 --> 04:30:19,960 TREATABLE NOW IT STARTED OUT 6160 04:30:19,960 --> 04:30:22,120 WITH DRUGS THAT MAY BE HAD A 6161 04:30:22,120 --> 04:30:23,360 TWO-MONTH AFFECT AND ANOTHER 6162 04:30:23,360 --> 04:30:25,560 ADDED ON AND NOW YOU'RE UP TO 6163 04:30:25,560 --> 04:30:30,800 FIVE MONTHS AND KEEP ADDING 6164 04:30:30,800 --> 04:30:33,360 UNTIL IT'S CURED AND ALS I THINK 6165 04:30:33,360 --> 04:30:36,480 IS A MISPRESCRIBED ILLNESS RIGHT 6166 04:30:36,480 --> 04:30:37,040 NOW AND GOING DOWN THE SAME 6167 04:30:37,040 --> 04:30:47,160 PATH. 6168 04:30:58,080 --> 04:31:01,160 >>LOOKING AT BIOLOGICAL 6169 04:31:01,160 --> 04:31:05,200 SIGNATURES CAN WE LEARN FROM 6170 04:31:05,200 --> 04:31:15,440 HAVING A BROADER HETROGENEIC 6171 04:31:15,440 --> 04:31:15,800 SUBGROUPS. 6172 04:31:15,800 --> 04:31:17,120 >>WE SHOULD NEVER BE LIMITED BY 6173 04:31:17,120 --> 04:31:22,760 OUR DESIGN OR BY POWER. 6174 04:31:22,760 --> 04:31:33,320 WE SHOULD NEVER HAVE TO HAVE TO 6175 04:31:45,240 --> 04:31:47,080 MAKE IT HARDER TO ASK QUESTIONS 6176 04:31:47,080 --> 04:31:49,000 YOU WANT TO ASK AND THERE'S WAY 6177 04:31:49,000 --> 04:31:51,480 TO ASK SMARTLY AND SHARE 6178 04:31:51,480 --> 04:31:54,600 INFORMATION ACROSS THE GROUPS 6179 04:31:54,600 --> 04:31:56,920 BUT LOOK AT THE HETEROGENEITY 6180 04:31:56,920 --> 04:31:58,520 ACROSS THEM AND IT BOILS DOWN TO 6181 04:31:58,520 --> 04:32:00,560 WHAT TIM MENTIONED IN OUR 6182 04:32:00,560 --> 04:32:02,960 PRIORITIES WHICH IS USING ALL OF 6183 04:32:02,960 --> 04:32:05,640 THE INFORMATION THAT WE HAVE AND 6184 04:32:05,640 --> 04:32:07,440 ALSO BUILDING SMARTER CLINICAL 6185 04:32:07,440 --> 04:32:09,280 TRIALS AND MORE POWERFUL 6186 04:32:09,280 --> 04:32:11,240 ANALYSIS METHODS AND BETTER 6187 04:32:11,240 --> 04:32:12,720 OUTCOME MEASURES, PUTTING ALL 6188 04:32:12,720 --> 04:32:14,240 THAT TOGETHER SO WE AREN'T 6189 04:32:14,240 --> 04:32:17,000 LIMITED BY THE THINGS AND WE CAN 6190 04:32:17,000 --> 04:32:20,480 DESIGN VERY POWERFUL TRIALS THAT 6191 04:32:20,480 --> 04:32:21,800 ENROLL A HOMOGENOUS SUBSET. 6192 04:32:21,800 --> 04:32:29,240 >>THANK YOU. 6193 04:32:29,240 --> 04:32:35,560 >>THE NEXT ONE IS FOR MERIT, 6194 04:32:35,560 --> 04:32:38,800 SHOULD A HORRIFYING FATAL 6195 04:32:38,800 --> 04:32:42,040 DISEASE LIKE ALS WITH NO CURE BE 6196 04:32:42,040 --> 04:32:47,560 GIVEN PRIORITY? 6197 04:32:47,560 --> 04:32:49,240 >>FIRST, I'LL CONFESS IN 6198 04:32:49,240 --> 04:32:50,840 CLINICAL BIAS I HAVE AND 6199 04:32:50,840 --> 04:32:51,440 EVERYBODY ON THE CALL HAS. 6200 04:32:51,440 --> 04:32:55,400 >>I WANTED TO ANSWER THIS ONE, 6201 04:32:55,400 --> 04:32:55,600 MERIT. 6202 04:32:55,600 --> 04:32:57,600 SAME REASON. 6203 04:32:57,600 --> 04:32:58,880 >>ABSOLUTELY. 6204 04:32:58,880 --> 04:33:01,040 I THINK NOT JUST AT THE NIH BUT 6205 04:33:01,040 --> 04:33:03,720 ALSO AT THE FDA AND I THINK 6206 04:33:03,720 --> 04:33:06,440 WE'RE SEEING THAT AND HEARING 6207 04:33:06,440 --> 04:33:09,320 THAT FROM THE FDA THAT THEY 6208 04:33:09,320 --> 04:33:10,880 UNDERSTAND THE SERIOUSNESS OF 6209 04:33:10,880 --> 04:33:12,480 THE ILLNESS AND THE FLEXIBILITY 6210 04:33:12,480 --> 04:33:14,560 NEEDED AND HOW TO PRIORITIZE AND 6211 04:33:14,560 --> 04:33:16,920 BY HAVING THE MEANING AND WE'RE 6212 04:33:16,920 --> 04:33:18,800 SEEING THAT FROM NIH AS WELL I 6213 04:33:18,800 --> 04:33:21,520 THINK THERE ARE A LOT OF 6214 04:33:21,520 --> 04:33:22,480 ILLNESSES NEUROLOGICAL ILLNESSES 6215 04:33:22,480 --> 04:33:24,240 THAT HAVE UNMET NEEDS BUT THIS 6216 04:33:24,240 --> 04:33:28,680 IS ONE OF THE MOST RAPID AND I'M 6217 04:33:28,680 --> 04:33:31,160 EXCITED ABOUT THE AMOUNT OF 6218 04:33:31,160 --> 04:33:32,080 SCIENCE HAPPENING. 6219 04:33:32,080 --> 04:33:33,760 IT DEFINITELY NEEDS TO BE 6220 04:33:33,760 --> 04:33:38,560 PRIORITIZED. 6221 04:33:38,560 --> 04:33:39,760 >>DOES ANYBODY FROM THE 6222 04:33:39,760 --> 04:33:41,640 COMMITTEE WANT TO WEIGH IN. 6223 04:33:41,640 --> 04:33:43,680 >>WE'LL ALL AGREE WITH THE IDEA 6224 04:33:43,680 --> 04:33:45,560 IT SHOULD BE A PRIORITY FOR LOTS 6225 04:33:45,560 --> 04:33:55,760 OF REASONS. 6226 04:33:56,480 --> 04:33:59,520 >>TO WEIGH IN ON HOW STRINGENT 6227 04:33:59,520 --> 04:34:00,760 CRITERIA NEED TO BE AND GET USE 6228 04:34:00,760 --> 04:34:01,760 OF SOMETHING AND THE QUESTION 6229 04:34:01,760 --> 04:34:06,200 ABOUT DO WE HAVE MODELS IN OTHER 6230 04:34:06,200 --> 04:34:09,760 DISEASES AND THIS IS A PLACE 6231 04:34:09,760 --> 04:34:11,120 WHERE WE DO. 6232 04:34:11,120 --> 04:34:14,640 IN GENERAL PEOPLE WITH CANCER AT 6233 04:34:14,640 --> 04:34:19,640 VIRTUALLY ALL PHASES CAN FIND A 6234 04:34:19,640 --> 04:34:20,600 PROTOCOL AND IMPORTANT CRUCIAL 6235 04:34:20,600 --> 04:34:22,600 INFORMATION CAN BE LEARNED BY 6236 04:34:22,600 --> 04:34:23,320 THEIR PARTICIPATION IN TRIALS 6237 04:34:23,320 --> 04:34:24,160 ALL THE WAY THROUGH THE GAMUT OF 6238 04:34:24,160 --> 04:34:27,320 THE DISEASE. 6239 04:34:27,320 --> 04:34:30,840 I THINK WE SHOULD BE VERY 6240 04:34:30,840 --> 04:34:32,800 EXPLICIT ABOUT OUR DESIRE FOR 6241 04:34:32,800 --> 04:34:33,600 ALS TRIALS IN THE FUTURE SHOULD 6242 04:34:33,600 --> 04:34:35,680 BE ANYBODY WHO WANTS TO BE IN 6243 04:34:35,680 --> 04:34:37,800 THE TRIAL SHOULD BE IN ONE AND I 6244 04:34:37,800 --> 04:34:40,960 DON'T REALLY MEAN EXPANDED 6245 04:34:40,960 --> 04:34:41,720 ACCESS NECESSARILY. 6246 04:34:41,720 --> 04:34:43,400 A TRIAL BY WHICH WE LEARN 6247 04:34:43,400 --> 04:34:44,040 SOMETHING THAT TAKES THE FIELD 6248 04:34:44,040 --> 04:34:54,200 FORWARD. 6249 04:34:55,520 --> 04:34:56,400 >>NEXT QUESTION MAYBE FOR THREE 6250 04:34:56,400 --> 04:34:58,920 OF OUR PANELISTS, JEREMY, 6251 04:34:58,920 --> 04:35:01,560 CHRISTINA AND COMBINING TWO 6252 04:35:01,560 --> 04:35:04,520 QUESTIONS AND ONE IS HOW CAN NIH 6253 04:35:04,520 --> 04:35:05,640 ENCOURAGE THE DEVELOPMENT OF 6254 04:35:05,640 --> 04:35:09,760 BETTER CLINICAL OUTCOME MEASURES 6255 04:35:09,760 --> 04:35:12,480 WITH HIGH REPRODUCIBILITY? 6256 04:35:12,480 --> 04:35:16,400 AND HOW DO WE ENCOURAGE MORE 6257 04:35:16,400 --> 04:35:17,720 DEVELOPMENT OF THE BIOMARKERS 6258 04:35:17,720 --> 04:35:20,320 AND THE LAST PART I'LL ADD IN IS 6259 04:35:20,320 --> 04:35:22,280 SOMETHING THAT CAME FROM THE Q&A 6260 04:35:22,280 --> 04:35:24,920 SHOULD WE BE MANDATING EVERY 6261 04:35:24,920 --> 04:35:27,160 TRIAL HAS THE GSF BIOMARKERS? 6262 04:35:27,160 --> 04:35:28,680 MAYBE JEREMY, IF YOU WANT TO GO 6263 04:35:28,680 --> 04:35:30,720 FIRST AND THEN CHRISTINA AND 6264 04:35:30,720 --> 04:35:31,800 THEN BOB. 6265 04:35:31,800 --> 04:35:37,480 >>WELL, I'LL DEFER ON THE FLUID 6266 04:35:37,480 --> 04:35:40,840 BIOMARKERS TO BOB AND JUST TALK 6267 04:35:40,840 --> 04:35:44,160 ABOUT ALS OUTCOME MEASURES MORE 6268 04:35:44,160 --> 04:35:44,400 BROADLY. 6269 04:35:44,400 --> 04:35:47,920 THERE IS A PERCEPTION THAT 6270 04:35:47,920 --> 04:35:49,600 CLINICAL TRIAL DEVELOPMENT IS 6271 04:35:49,600 --> 04:35:52,280 LIMITED BY THE QUALITY OF OUR 6272 04:35:52,280 --> 04:35:52,760 BIOMARKERS. 6273 04:35:52,760 --> 04:35:55,320 AND I'M REALLY NOT CONVINCED 6274 04:35:55,320 --> 04:35:56,040 THAT THE CLINICAL TRIALS THAT 6275 04:35:56,040 --> 04:36:00,800 WE'RE PERFORMING TODAY ARE SO 6276 04:36:00,800 --> 04:36:01,040 LIMITED. 6277 04:36:01,040 --> 04:36:03,560 I THINK WE HAVE A SCALE THAT'S 6278 04:36:03,560 --> 04:36:06,680 SHOWN TO BE SENSITIVE TO VERY 6279 04:36:06,680 --> 04:36:11,160 MODEST MODIFICATIONS BY A DRUG. 6280 04:36:11,160 --> 04:36:13,600 AND IT CLEAR METRIC DIFFICULTIES 6281 04:36:13,600 --> 04:36:16,080 AND CHRISTINA HAS DEVELOPED A 6282 04:36:16,080 --> 04:36:21,120 SCALE THAT MAY ACTUALLY ADDRESS 6283 04:36:21,120 --> 04:36:24,840 A GOOD NUMBER OF THESE 6284 04:36:24,840 --> 04:36:25,760 WEAKNESSES BUT NONETHELESS IT 6285 04:36:25,760 --> 04:36:27,200 DOES A GOOD JOB OF SHOWING 6286 04:36:27,200 --> 04:36:28,840 MODEST AFFECTS AND WHEN IT DOES 6287 04:36:28,840 --> 04:36:30,600 SHOW AN EFFECT, IT SHOWS AN 6288 04:36:30,600 --> 04:36:32,720 AFFECT THAT IS MIRRORED BY 6289 04:36:32,720 --> 04:36:34,360 EVERYTHING ELSE WE DO INCLUDING 6290 04:36:34,360 --> 04:36:35,920 EVALUATIONS OF STRENGTH, 6291 04:36:35,920 --> 04:36:39,680 EVALUATION MUCH BREEDING -- 6292 04:36:39,680 --> 04:36:40,920 BREATHING AND QUALITY OF LIFE. 6293 04:36:40,920 --> 04:36:47,280 MY VIEW OF WHAT NEEDS TO BE DONE 6294 04:36:47,280 --> 04:36:48,280 IN THE FUTURE INCLUDES NOT ONLY 6295 04:36:48,280 --> 04:36:50,240 MAKING A FUNCTIONAL RATING SCALE 6296 04:36:50,240 --> 04:36:51,680 BETTER BUT TO THINK ABOUT WHAT 6297 04:36:51,680 --> 04:36:54,480 PARTS OF ALS DON'T WE ADDRESS? 6298 04:36:54,480 --> 04:36:57,400 AND THERE ARE A NUMBER OF THEM. 6299 04:36:57,400 --> 04:37:01,040 ALS IS A DISEASE OF CENTRAL AND 6300 04:37:01,040 --> 04:37:03,440 PERIPHERAL MOTOR NEURONS. 6301 04:37:03,440 --> 04:37:05,600 WE EVALUATE THEM PRETTY POORLY 6302 04:37:05,600 --> 04:37:08,480 AND THAT MAY NOT DIRECTLY HELP 6303 04:37:08,480 --> 04:37:09,160 OUR CLINICAL TRIAL INVESTIGATION 6304 04:37:09,160 --> 04:37:10,800 BUT HELP LEARN ABOUT THE DISEASE 6305 04:37:10,800 --> 04:37:11,400 IF WE CAN INVESTIGATE THAT 6306 04:37:11,400 --> 04:37:15,680 BETTER. 6307 04:37:15,680 --> 04:37:21,760 THERE IS A CLEAR OVERLAP BETWEEN 6308 04:37:21,760 --> 04:37:29,440 FRONTAL TEMPORAL DEMENTIA AND WE 6309 04:37:29,440 --> 04:37:32,680 EVALUATE COGNITIVE DEFICIENCIES 6310 04:37:32,680 --> 04:37:34,760 POORLY AND WE TREAT THEM BETTER 6311 04:37:34,760 --> 04:37:38,160 WHEN WE CAN MEASURE THEM BETTER. 6312 04:37:38,160 --> 04:37:38,960 THAT'S THE DIRECTION THAT I 6313 04:37:38,960 --> 04:37:44,000 WOULD LIKE TO SEE US GO AS TO 6314 04:37:44,000 --> 04:37:45,760 FOCUS ON WHAT WE KNOW WE'RE NOT 6315 04:37:45,760 --> 04:37:47,360 MEASURING IN ALS AND FOCUS ON 6316 04:37:47,360 --> 04:37:48,600 WHY THAT'S IMPORTANT AND WHAT WE 6317 04:37:48,600 --> 04:37:50,600 CAN DO BETTER AND HOW DO WE 6318 04:37:50,600 --> 04:37:57,760 MEASURE THAT IMPROVEMENT AND WE 6319 04:37:57,760 --> 04:37:59,080 KCAN DO THAT. 6320 04:37:59,080 --> 04:38:00,960 >>THERE'S AN OPPORTUNITY TO 6321 04:38:00,960 --> 04:38:04,200 TACK ON TO EXISTING TRIALS AND 6322 04:38:04,200 --> 04:38:05,240 EXISTING NATURAL HISTORY EFFORTS 6323 04:38:05,240 --> 04:38:05,920 AND NOVEL THINGS TO ADVANCE THEM 6324 04:38:05,920 --> 04:38:15,560 QUICKER. 6325 04:38:15,560 --> 04:38:19,160 NIH CAN SUPPORT ADJUNCT MEASURES 6326 04:38:19,160 --> 04:38:22,040 TO LEARN MORE ABOUT BIOMARKERS 6327 04:38:22,040 --> 04:38:25,760 AND NOVEL OUTCOMES EVEN IF THE 6328 04:38:25,760 --> 04:38:28,320 STUDY IS NEGATIVE APPROACHING 6329 04:38:28,320 --> 04:38:29,840 EACH STUDY MAKING SURE WE LEARN 6330 04:38:29,840 --> 04:38:33,720 SOMETHING WHETHER IT'S HOW TO 6331 04:38:33,720 --> 04:38:34,720 BETTER MEASURE DISEASE OR 6332 04:38:34,720 --> 04:38:37,040 SOMETHING NEW TO MAKE SURE EVERY 6333 04:38:37,040 --> 04:38:37,920 STUDY CONTRIBUTE TO MOVING 6334 04:38:37,920 --> 04:38:39,560 FORWARD EVEN IF IT'S NOT A 6335 04:38:39,560 --> 04:38:40,280 POSITIVE DRUG. 6336 04:38:40,280 --> 04:38:41,680 THIS IS ONE PLACE WHERE ACADEMIC 6337 04:38:41,680 --> 04:38:44,760 AND INDUSTRY PARTNERSHIPS ARE 6338 04:38:44,760 --> 04:38:47,800 CRITICAL AND NIH PARTNERSHIPS TO 6339 04:38:47,800 --> 04:38:49,560 LEVERAGE WHAT WE'RE ALREADY 6340 04:38:49,560 --> 04:38:50,520 DOING TO LEARN MORE AND ADVANCE 6341 04:38:50,520 --> 04:39:00,760 OUR OUTCOMES. 6342 04:39:06,720 --> 04:39:09,560 >>THERE WAS A TALK OF ABOUT 6343 04:39:09,560 --> 04:39:10,880 LUMBAR TAPS INCLUSION IN THE 6344 04:39:10,880 --> 04:39:12,840 TRIALS AND THINK WE NEED MORE 6345 04:39:12,840 --> 04:39:15,560 THAN ONE AND SHOULD BE. 6346 04:39:15,560 --> 04:39:18,600 IN SOME OF THE RATIONALE 6347 04:39:18,600 --> 04:39:20,440 INVOLVES A QUESTION AROUND 6348 04:39:20,440 --> 04:39:22,080 PHARMACO DYNAMIC BIOMARKERS. 6349 04:39:22,080 --> 04:39:23,280 IF YOU WANT TO GET THE FEEL IF 6350 04:39:23,280 --> 04:39:29,680 THE DRUG IS HITTING THE TARGET 6351 04:39:29,680 --> 04:39:34,960 IN THE CENTRAL NERVOUS SYSTEM 6352 04:39:34,960 --> 04:39:37,080 YOU NEED TO ADD LUMBAR PUNCTURES 6353 04:39:37,080 --> 04:39:39,080 AT THE BEGINNING, BASELINE AND 6354 04:39:39,080 --> 04:39:40,400 TOWARDS THE END OF THE TREATMENT 6355 04:39:40,400 --> 04:39:43,080 IN ORDER TO DEMONSTRATE THAT BUT 6356 04:39:43,080 --> 04:39:44,760 THE MORE THE BETTER IF YOU ASK 6357 04:39:44,760 --> 04:39:52,120 ME. 6358 04:39:52,120 --> 04:39:54,040 ANOTHER QUESTION WAS AROUND 6359 04:39:54,040 --> 04:39:54,720 REPRODUCIBILITY AND RELIABILITY 6360 04:39:54,720 --> 04:39:57,080 AND MEASUREMENTS, ETCETERA. 6361 04:39:57,080 --> 04:39:59,680 ONE AREA I THINK NIH COULD 6362 04:39:59,680 --> 04:40:02,520 REALLY PARTICIPATE AND TAKE A 6363 04:40:02,520 --> 04:40:05,240 LEAD IN SPONSORING IS SOME OF 6364 04:40:05,240 --> 04:40:07,560 THE APPROACHES THAT HAVE BEEN 6365 04:40:07,560 --> 04:40:09,760 INCLUDED IN THE FEW BUT HAVE 6366 04:40:09,760 --> 04:40:12,080 BEEN MAINLY OUT OF OR WITH OUR 6367 04:40:12,080 --> 04:40:15,720 EUROPEAN COLLEAGUES AND THAT IS 6368 04:40:15,720 --> 04:40:18,280 REALLY DOING MORE ASSAY 6369 04:40:18,280 --> 04:40:20,120 VALIDATION FOR WHATEVER A 6370 04:40:20,120 --> 04:40:20,760 PARTICULAR BIOMARKER IS. 6371 04:40:20,760 --> 04:40:25,760 SO YOU LOOK AT THEM AND SAY LIKE 6372 04:40:25,760 --> 04:40:27,440 ROUND ROBIN TYPES OF STUDIES 6373 04:40:27,440 --> 04:40:28,760 HARD TO GET FUNDING TO DO. 6374 04:40:28,760 --> 04:40:30,800 NO ONE WANTS TO FUND IT. 6375 04:40:30,800 --> 04:40:33,240 AND SO IT'S A CHALLENGE. 6376 04:40:33,240 --> 04:40:34,720 IF YOU HAVE A BIOMARKER OF 6377 04:40:34,720 --> 04:40:35,640 INTEREST AND DEVELOP A 6378 04:40:35,640 --> 04:40:41,080 PARTICULAR ASSAY FOR IT, BEING 6379 04:40:41,080 --> 04:40:44,000 ABLE TO INCLUDE A NUMBER OF 6380 04:40:44,000 --> 04:40:45,480 SITES THAT ALL RUN THE SAME 6381 04:40:45,480 --> 04:40:47,240 SAMPLES AND ASSAY AND 6382 04:40:47,240 --> 04:40:48,880 REPRODUCING THE RELIABILITY OF 6383 04:40:48,880 --> 04:40:51,240 THE SIGNAL IS CRITICAL TO MOVING 6384 04:40:51,240 --> 04:40:52,440 ANY BIOMARKER FORWARD. 6385 04:40:52,440 --> 04:40:55,040 WE'RE AT A POINT WHERE WE HAVE A 6386 04:40:55,040 --> 04:40:57,600 HOST OF BIOMARKERS THAT LOOK 6387 04:40:57,600 --> 04:40:57,920 INTERESTING. 6388 04:40:57,920 --> 04:41:00,200 SOME FOR SPECIFIC FORMS OF ALS 6389 04:41:00,200 --> 04:41:02,600 AND SOME FOR THE MORE SPORADIC 6390 04:41:02,600 --> 04:41:05,680 POPULATION AND ONE AREA NIH CAN 6391 04:41:05,680 --> 04:41:09,240 HELP MOVE THE NEEDLE IS TO FUND 6392 04:41:09,240 --> 04:41:09,840 THE STUDIES THAT DEMONSTRATE 6393 04:41:09,840 --> 04:41:15,240 BIOMARKER VALIDATION. 6394 04:41:15,240 --> 04:41:17,120 >>THANK YOU. 6395 04:41:17,120 --> 04:41:23,840 THIS NEXT ONE IS FOR CHIADI SO 6396 04:41:23,840 --> 04:41:26,840 QUESTION IS I WOULD LOVE TO SEE 6397 04:41:26,840 --> 04:41:30,920 ONE OF THESE TALENTED GROUPS 6398 04:41:30,920 --> 04:41:35,760 STUDY PATIENTS WHO ARE DOING 6399 04:41:35,760 --> 04:41:38,920 UNEXPECTEDLY WELL WITH THE 6400 04:41:38,920 --> 04:41:41,200 DISEASE, FOR EXAMPLE, THOSE WITH 6401 04:41:41,200 --> 04:41:43,440 SLOW PROGRESSION, LONG PLATEAUS 6402 04:41:43,440 --> 04:41:48,680 OR EVEN APPARENT RECOVERY FROM A 6403 04:41:48,680 --> 04:41:49,760 DISEASE. 6404 04:41:49,760 --> 04:41:53,320 STUDYING PATIENTS WHO ARE DONE 6405 04:41:53,320 --> 04:41:57,560 EXPECTEDLY WELL WITH OTHER 6406 04:41:57,560 --> 04:41:58,120 DISEASES HAS LED TO BETTER 6407 04:41:58,120 --> 04:42:03,960 TREATMENTS. 6408 04:42:03,960 --> 04:42:14,320 IE, HIV CONTROLLERS. 6409 04:42:19,120 --> 04:42:20,480 >>THE CHALLENGE IS IDENTIFYING 6410 04:42:20,480 --> 04:42:24,960 THE SPECIFIC NUMBER TO DO THIS. 6411 04:42:24,960 --> 04:42:30,920 FOR EXAMPLE IN THE FRONTAL 6412 04:42:30,920 --> 04:42:32,200 DEMENTIA FIELD THEY LOOK LIKE 6413 04:42:32,200 --> 04:42:33,720 THEY HAVE THE VARIANT BUT NOT 6414 04:42:33,720 --> 04:42:37,760 DECLINING AND OFTEN DO NOT HAVE 6415 04:42:37,760 --> 04:42:39,800 THE MRI FROM THE 6416 04:42:39,800 --> 04:42:40,640 NEUROPSYCHOLOGICAL PROFILES YOU 6417 04:42:40,640 --> 04:42:42,200 EXPECT FROM THE CLASSICALLY 6418 04:42:42,200 --> 04:42:44,080 DESCRIBED CONDITIONS BUT WITH 6419 04:42:44,080 --> 04:42:47,680 THE ADVENT AND DISCOVERY AND 6420 04:42:47,680 --> 04:42:49,760 GENETICS WE WERE THEN ABLE TO 6421 04:42:49,760 --> 04:42:54,880 SHOW SOME ARE MUTATION CARRIERS 6422 04:42:54,880 --> 04:42:57,840 AND MADE IT CLEAR WHAT WAS 6423 04:42:57,840 --> 04:43:00,000 VIEWED AS THE TERM PHENOCOPY 6424 04:43:00,000 --> 04:43:03,800 SUGGESTS THEY'RE MIMICS OPPOSED 6425 04:43:03,800 --> 04:43:06,680 TO THE ACTUAL THING. 6426 04:43:06,680 --> 04:43:08,560 BY BEING ABLE TO DEMONSTRATE 6427 04:43:08,560 --> 04:43:10,480 SOME OF THE LIMITATION CARRIERS 6428 04:43:10,480 --> 04:43:13,000 AND DEMONSTRATE AT LEAST IN 6429 04:43:13,000 --> 04:43:14,680 THOSE WHO PASSED EARLY THEY HAVE 6430 04:43:14,680 --> 04:43:17,600 CORRESPONDING PATHOLOGY WE WERE 6431 04:43:17,600 --> 04:43:18,480 ABLE TO IDENTIFY ESSENTIALLY 6432 04:43:18,480 --> 04:43:20,440 THAT THERE ARE MORE THAN ONE 6433 04:43:20,440 --> 04:43:23,400 TRAJECTORY OF PROGRESSION. 6434 04:43:23,400 --> 04:43:29,600 SO THEY'RE VERY SLOW 6435 04:43:29,600 --> 04:43:29,880 PROGRESSERS. 6436 04:43:29,880 --> 04:43:32,880 WHAT IS NEXT NEEDED IS 6437 04:43:32,880 --> 04:43:34,600 UNDERSTAND WHAT GOVERNS 6438 04:43:34,600 --> 04:43:35,320 HETEROGENEITY OF EXPRESSION. 6439 04:43:35,320 --> 04:43:38,320 FOR EXAMPLE, IF WE GO BACK TO 6440 04:43:38,320 --> 04:43:40,960 FTD AND ALS, WHY DO SOME PEOPLE 6441 04:43:40,960 --> 04:43:44,640 DEVELOP ALS AND SOME DEVELOP FTD 6442 04:43:44,640 --> 04:43:45,760 OR BOTH? 6443 04:43:45,760 --> 04:43:49,760 THERE'S A NUMBER OF QUESTIONS A 6444 04:43:49,760 --> 04:43:55,120 NUMBER OF QUESTIONS HOW AND WHEN 6445 04:43:55,120 --> 04:44:05,120 DISEASE IS EXPRESSED AND WE 6446 04:44:05,120 --> 04:44:09,560 COULD PROVIDE AN AVENUE FOR CURE 6447 04:44:09,560 --> 04:44:10,120 IF WE HAVE MORE INFORMATION. 6448 04:44:10,120 --> 04:44:13,240 >>THE NEXT QUESTION I THINK 6449 04:44:13,240 --> 04:44:16,320 ALSO, CHIADI AND MELANIE ALSO, 6450 04:44:16,320 --> 04:44:19,560 THERE'S A QUESTION BY WHETHER 6451 04:44:19,560 --> 04:44:22,480 EXERCISE IN DIET MAY CONTRIBUTE 6452 04:44:22,480 --> 04:44:24,120 TO ALS PROGRESSION WE KNOW MAKES 6453 04:44:24,120 --> 04:44:30,080 A HUGE DIFFERENCE IN AGING AND 6454 04:44:30,080 --> 04:44:30,880 IN OTHER NEURODEGENERATIVE 6455 04:44:30,880 --> 04:44:32,000 DISEASES HAVE SHOWN TO GET 6456 04:44:32,000 --> 04:44:35,360 BETTER WITH EXERCISE. 6457 04:44:35,360 --> 04:44:39,440 ARE THERE WAYS TO STUDY THIS 6458 04:44:39,440 --> 04:44:41,240 WHETHER IT'S TRIALS OR OTHER 6459 04:44:41,240 --> 04:44:43,080 APPROACHES WHETHER EXERCISE AND 6460 04:44:43,080 --> 04:44:44,160 DIET CAN MAKE A DIFFERENCE? 6461 04:44:44,160 --> 04:44:50,400 >>IT'S A PRIORITY TO UNDERSTAND 6462 04:44:50,400 --> 04:44:52,680 WA THE SOCIAL CULTURAL FACTORS 6463 04:44:52,680 --> 04:44:54,560 ARE THAT AFFECT DISEASE 6464 04:44:54,560 --> 04:44:55,600 PROGRESSION AND GOES BEYOND 6465 04:44:55,600 --> 04:44:59,200 EXERCISE AND DIET TO OTHER 6466 04:44:59,200 --> 04:45:02,800 SOCIAL PRACTICES, SOCIALIZATION 6467 04:45:02,800 --> 04:45:04,720 IN GENERAL, FOR EXAMPLE. 6468 04:45:04,720 --> 04:45:07,960 IT SEEMS CLEAR THAT DIET IN 6469 04:45:07,960 --> 04:45:11,680 PARTICULAR, NUTRITIONAL STATUS 6470 04:45:11,680 --> 04:45:16,840 AFFECTS ALS OUTCOMES AND WHAT 6471 04:45:16,840 --> 04:45:21,160 MAYBE IS LESS CLEAR IS TO WHAT 6472 04:45:21,160 --> 04:45:22,040 EXTENT DIET MAY DETERMINE ALS IN 6473 04:45:22,040 --> 04:45:23,720 THE FIRST PLACE AND THEN OF 6474 04:45:23,720 --> 04:45:29,720 COURSE YOU WANT TO KNOW TO WHAT 6475 04:45:29,720 --> 04:45:35,680 EXTENT IT'S MODIFIABLE. 6476 04:45:35,680 --> 04:45:39,040 NATURAL HISTORY STUDIES DEVELOP 6477 04:45:39,040 --> 04:45:40,560 ANSWERS TO THE QUESTIONS. 6478 04:45:40,560 --> 04:45:43,800 >>MELANIE, I DIDN'T KNOW IF YOU 6479 04:45:43,800 --> 04:45:46,320 HAD ANYTHING TO ADD OF WAYS TO 6480 04:45:46,320 --> 04:45:47,560 INCORPORATE IT INTO TRIALS. 6481 04:45:47,560 --> 04:45:49,680 >>WE DO A LOT OF TRIALS THAT 6482 04:45:49,680 --> 04:45:51,120 HAVE MULTIPLE DOMAINS IN WHICH 6483 04:45:51,120 --> 04:45:51,720 WE LOOK AT DIFFERENT TYPES OF 6484 04:45:51,720 --> 04:45:59,920 THINGS. 6485 04:45:59,920 --> 04:46:01,760 AND LOOKING AT THE PLATFORM 6486 04:46:01,760 --> 04:46:05,720 TRIAL IN WHICH WE'RE STUDYING 6487 04:46:05,720 --> 04:46:09,240 DIFFERENT THERAPEUTICS 6488 04:46:09,240 --> 04:46:11,720 INTERVENT 6489 04:46:11,720 --> 04:46:14,480 INTERVENTIONS LOOKING AT DIET 6490 04:46:14,480 --> 04:46:16,040 DOMAINS OR EXERCISE DOMAINS TO 6491 04:46:16,040 --> 04:46:17,720 UNDERSTAND WHICH INTERACTION OF 6492 04:46:17,720 --> 04:46:21,360 ALL THE THINGS LEADS TO THE BEST 6493 04:46:21,360 --> 04:46:24,640 POSSIBLE OUTCOME. 6494 04:46:24,640 --> 04:46:26,480 AND THERE'S A LOT THAT'S BEEN 6495 04:46:26,480 --> 04:46:31,440 DONE IN TERMS OF COVID IN 6496 04:46:31,440 --> 04:46:32,120 PLATFORM TRIALS. 6497 04:46:32,120 --> 04:46:33,760 AND THERE'S ROOM TO ADD THOSE 6498 04:46:33,760 --> 04:46:43,080 INTO TRIALS. 6499 04:46:43,080 --> 04:46:45,200 >>DO WE HAVE TIME FOR ONE MORE 6500 04:46:45,200 --> 04:46:45,560 QUESTION? 6501 04:46:45,560 --> 04:46:49,480 >>I THINK SO, YEAH. 6502 04:46:49,480 --> 04:46:55,480 I THOUGHT WE HAD UNTIL 3:44. 6503 04:46:55,480 --> 04:46:59,600 >>WE HAVE GOT TIME. 6504 04:46:59,600 --> 04:47:10,000 SO THIS ONE'S FOR BOB. 6505 04:47:11,800 --> 04:47:13,600 MY PERSON WITH ALS IS NEWLY 6506 04:47:13,600 --> 04:47:14,640 DIAGNOSED IN THE REGISTRY. 6507 04:47:14,640 --> 04:47:21,120 SO FAR IT SEEMS THE ONUS IS ON 6508 04:47:21,120 --> 04:47:24,440 THE PATIENT TO REACH OUT AND 6509 04:47:24,440 --> 04:47:25,760 SEARCH FOR STUDIES. 6510 04:47:25,760 --> 04:47:27,200 I CAN RELATE. 6511 04:47:27,200 --> 04:47:29,120 ARE THERE TRIAL-MATCHING TOOLS 6512 04:47:29,120 --> 04:47:35,760 UNDER DEVELOPMENT TO CONNECT 6513 04:47:35,760 --> 04:47:38,280 WITH POTENTIAL STUDIES AND/OR 6514 04:47:38,280 --> 04:47:39,240 BIO SAMPLE COLLECTION. 6515 04:47:39,240 --> 04:47:42,200 I'LL STOP THERE. 6516 04:47:42,200 --> 04:47:43,880 >>WOW, THAT'S A GREAT QUESTION. 6517 04:47:43,880 --> 04:47:47,680 I'M ACTUALLY NOT SURE IF THERE 6518 04:47:47,680 --> 04:47:49,440 ARE TOOLS BEING DEVELOPED TO GO 6519 04:47:49,440 --> 04:47:51,760 THE OPPOSITE DIRECTION OF WHAT 6520 04:47:51,760 --> 04:47:54,360 YOU JUST SAID, GWEN, TRYING TO 6521 04:47:54,360 --> 04:47:58,440 TAKE AND IDENTIFY INDIVIDUALS 6522 04:47:58,440 --> 04:48:00,600 FROM EITHER LISTINGS OR OTHER 6523 04:48:00,600 --> 04:48:03,320 AREAS IN ORDER TO IDENTIFY THOSE 6524 04:48:03,320 --> 04:48:04,720 THAT MIGHT BE BEST ENROLLED IN A 6525 04:48:04,720 --> 04:48:08,960 PARTICULAR STUDY OR TRIAL. 6526 04:48:08,960 --> 04:48:11,360 I KNOW THERE ARE A FEW GROUPS 6527 04:48:11,360 --> 04:48:13,760 THAT ARE QUITE GOOD AT LISTING 6528 04:48:13,760 --> 04:48:15,200 ALL THE VARIOUS TRIALS AND 6529 04:48:15,200 --> 04:48:17,800 RESEARCH STUDIES THAT ARE 6530 04:48:17,800 --> 04:48:18,040 AVAILABLE. 6531 04:48:18,040 --> 04:48:21,760 BUT AGAIN I'M NOT REALLY -- I'M 6532 04:48:21,760 --> 04:48:23,720 NOT KNOWLEDGEABLE ABOUT ANY 6533 04:48:23,720 --> 04:48:27,240 KNOWN ILLNESS OR ALGORITHM THAT 6534 04:48:27,240 --> 04:48:28,080 WOULD TAKE INFORMATION AROUND AN 6535 04:48:28,080 --> 04:48:31,320 INDIVIDUAL PATIENT AND IDENTIFY 6536 04:48:31,320 --> 04:48:34,320 WHAT ARE THE BEST OR OPPORTUNE 6537 04:48:34,320 --> 04:48:35,720 STUDIES OR TRIALS AVAILABLE FOR 6538 04:48:35,720 --> 04:48:37,760 THAT INDIVIDUAL. 6539 04:48:37,760 --> 04:48:39,640 I'M THROW IT TO THE OTHER 6540 04:48:39,640 --> 04:48:45,400 PANELISTS IF ANYONE'S AWARE OF 6541 04:48:45,400 --> 04:48:46,720 SUCH A TOOL. 6542 04:48:46,720 --> 04:48:49,600 >>I'M GOING TO JUMP IN ABOUT 6543 04:48:49,600 --> 04:48:54,800 THE CDC AND MAYBE PASS ITS -- IT 6544 04:48:54,800 --> 04:48:58,400 TO MERIT. 6545 04:48:58,400 --> 04:49:05,240 THE CDC WILL ADVERTISE FOR 6546 04:49:05,240 --> 04:49:05,480 TRIALS. 6547 04:49:05,480 --> 04:49:16,000 SO THEY DO AND WE CAN HELP WITH 6548 04:49:17,160 --> 04:49:18,280 CONNECTING PEOPLE AND THERE'S 6549 04:49:18,280 --> 04:49:18,920 INCUMBENT ON THE PERSON 6550 04:49:18,920 --> 04:49:19,520 CONTACTED TO REACH OUT TO THE 6551 04:49:19,520 --> 04:49:26,880 TRIAL. 6552 04:49:26,880 --> 04:49:27,040 >> 6553 04:49:27,040 --> 04:49:37,400 >>IT'S A GOOD IDEA. 6554 04:49:41,480 --> 04:49:42,200 AND THERE'S A CLINICAL TRIAL 6555 04:49:42,200 --> 04:49:45,760 MATCH MAKING TOOL. 6556 04:49:45,760 --> 04:49:49,560 THE NEED IS THERE AND THERE'S 6557 04:49:49,560 --> 04:49:53,760 ENOUGH TRIALS TO DO THIS. 6558 04:49:53,760 --> 04:49:57,200 THANK YOU. 6559 04:49:57,200 --> 04:49:58,040 >>NEIL'S WEBSITE HAS 6560 04:49:58,040 --> 04:49:59,040 INFORMATION ABOUT TRIALS AND 6561 04:49:59,040 --> 04:50:03,040 THREES A TRIAL NAVIGATOR PEOPLE 6562 04:50:03,040 --> 04:50:04,600 CAN CONTACT AS A PERSON THAT 6563 04:50:04,600 --> 04:50:10,680 WILL HELP THEM NAVIGATE AND FIND 6564 04:50:10,680 --> 04:50:11,720 TRIALS. 6565 04:50:11,720 --> 04:50:20,640 THOSE PEOPLE EXIST AND THERE'S 6566 04:50:20,640 --> 04:50:24,200 WAYS TO MAKE THE MATCH EASIER 6567 04:50:24,200 --> 04:50:29,760 AND ALGORITHM MAY BE BETTER BUT 6568 04:50:29,760 --> 04:50:35,000 ALSO MAYBE PROBLEMATIC. 6569 04:50:35,000 --> 04:50:35,760 >>THERE'S PATIENT INVOLVEMENT 6570 04:50:35,760 --> 04:50:37,280 AND TRYING TO GET THE NAVIGATION 6571 04:50:37,280 --> 04:50:39,240 AND INFORMATION THAT WILL BEST 6572 04:50:39,240 --> 04:50:41,720 HELP THEM DECIDE WHAT TRIAL THEY 6573 04:50:41,720 --> 04:50:52,040 WISH TO GET INTO. 6574 04:50:53,800 --> 04:50:55,200 >>DO WE HAVE ENOUGH SCIENTISTS 6575 04:50:55,200 --> 04:50:56,320 TO DO THE WORK? 6576 04:50:56,320 --> 04:50:57,400 IF NOT WHAT RECOMMENDATIONS DO 6577 04:50:57,400 --> 04:51:00,360 YOU HAVE TO ENSURE WE DO? 6578 04:51:00,360 --> 04:51:02,320 I HAVE THOUGHTS ON IT TOO BUT 6579 04:51:02,320 --> 04:51:03,640 OTHER COMMITTEE MEMBERS GO 6580 04:51:03,640 --> 04:51:03,840 FIRST. 6581 04:51:03,840 --> 04:51:14,360 >>I CAN START IF YOU'D LIKE. 6582 04:51:15,560 --> 04:51:18,160 >>SOMETIMES I THINK YES AND 6583 04:51:18,160 --> 04:51:19,680 SOMETIMES NO AND WE NEED MORE 6584 04:51:19,680 --> 04:51:22,800 PEOPLE COMING THROUGH WITH THE 6585 04:51:22,800 --> 04:51:28,800 SKILL SETS AND HAVE PLACES 6586 04:51:28,800 --> 04:51:30,200 PEOPLE CAN GET TRAINED. 6587 04:51:30,200 --> 04:51:34,600 THE FIX FOR THAT IS TO ENABLE A 6588 04:51:34,600 --> 04:51:37,600 TRAINING PROGRAM FOCUSSED ON ALS 6589 04:51:37,600 --> 04:51:39,400 OR MAYBE CLINICAL SCIENTIST 6590 04:51:39,400 --> 04:51:41,320 TRAINING MORE BROADLY. 6591 04:51:41,320 --> 04:51:45,440 I THINK THERE IS A NEED AS 6592 04:51:45,440 --> 04:51:47,720 HIGHLIGHTED BY THE QUESTION. 6593 04:51:47,720 --> 04:51:49,760 >>DO OTHER PEOPLE WANT TO WEIGH 6594 04:51:49,760 --> 04:51:50,480 IN ON THE QUESTION? 6595 04:51:50,480 --> 04:51:52,480 >>I'LL THROW IT THE OPPOSITE 6596 04:51:52,480 --> 04:51:53,400 DIRECTION. 6597 04:51:53,400 --> 04:51:55,520 SO I THINK AS YOU JUST SAID, 6598 04:51:55,520 --> 04:51:59,720 TIM, I THINK THERE'S A NEED FOR 6599 04:51:59,720 --> 04:52:02,360 MORE ALS NEUROMUSCULAR FOCUS 6600 04:52:02,360 --> 04:52:02,880 CLINICIAN SCIENTISTS. 6601 04:52:02,880 --> 04:52:06,760 YOU CAN ALSO GO THE OTHER 6602 04:52:06,760 --> 04:52:12,800 DIRECTION AND THAT IS HAVE A 6603 04:52:12,800 --> 04:52:16,760 Ph.D. LEVEL NEUROSCIENTIST THAT 6604 04:52:16,760 --> 04:52:18,040 WORKS OUT WITH CLINICAL 6605 04:52:18,040 --> 04:52:19,000 COLLEAGUES AT ACADEMIC 6606 04:52:19,000 --> 04:52:20,840 INSTITUTIONS OR OTHER SITES THAT 6607 04:52:20,840 --> 04:52:21,880 HELP DRIVE AND PUSH THE 6608 04:52:21,880 --> 04:52:24,840 GENERATION OF NEW STUDIES. 6609 04:52:24,840 --> 04:52:29,720 SO I THINK WE CAN WORK BETTER AT 6610 04:52:29,720 --> 04:52:30,600 PROVIDING OPPORTUNITIES FOR THE 6611 04:52:30,600 --> 04:52:33,040 Ph.D. NEURO SCIENTISTS TO 6612 04:52:33,040 --> 04:52:34,320 INTERACT WITH COLLEAGUES TO HELP 6613 04:52:34,320 --> 04:52:36,040 FACILITATE THE GENERATION OF NEW 6614 04:52:36,040 --> 04:52:39,160 RESEARCH STUDIES IN THE AREA OF 6615 04:52:39,160 --> 04:52:39,720 ALS. 6616 04:52:39,720 --> 04:52:41,760 >>IF I MAY WEIGH IN AS WELL. 6617 04:52:41,760 --> 04:52:42,920 THERE'S OPPORTUNITIES TO 6618 04:52:42,920 --> 04:52:44,440 COLLABORATE WITH PEOPLE IN 6619 04:52:44,440 --> 04:52:45,680 SCHOOLS OF PUBLIC HEALTH WHETHER 6620 04:52:45,680 --> 04:52:49,520 THEY'RE ACCUSTOMED TO DOING 6621 04:52:49,520 --> 04:52:51,280 LARGE-SCALE CLINICAL TRIALS. 6622 04:52:51,280 --> 04:52:55,320 IN THE EARLY DAYS OF DEMENTIA 6623 04:52:55,320 --> 04:52:56,680 CLINICAL TRIALS IT WAS A COMMON 6624 04:52:56,680 --> 04:52:58,720 PRACTICE TO HAVE WHAT I CALL 6625 04:52:58,720 --> 04:53:03,400 COORDINATING UNDER THES USUALLY 6626 04:53:03,400 --> 04:53:06,480 BASED IN SCHOOLS IN EPIDEMIOLOGY 6627 04:53:06,480 --> 04:53:10,480 AND STATISTICS AND THE PEOPLE IN 6628 04:53:10,480 --> 04:53:16,560 THE ALS FIELD COULD PARTNER IN 6629 04:53:16,560 --> 04:53:18,040 THE IMPLEMENTATION OF CLINICAL 6630 04:53:18,040 --> 04:53:21,760 TRIALS TO GET MORE DONE EVEN 6631 04:53:21,760 --> 04:53:26,440 WITHOUT HAVING TO TRAIN UP THAT 6632 04:53:26,440 --> 04:53:27,440 MANY PEOPLE. 6633 04:53:27,440 --> 04:53:33,080 >>AND FOR THE RECOVERING 6634 04:53:33,080 --> 04:53:36,640 NEUROLOGY CHAIR, IT'S A 6635 04:53:36,640 --> 04:53:37,280 MULTI-FACTORIAL PROBLEM AND 6636 04:53:37,280 --> 04:53:39,400 ACROSS THE C STATE IT'S HARD TO 6637 04:53:39,400 --> 04:53:41,000 FIT THIS INTO EITHER AN ACADEMIC 6638 04:53:41,000 --> 04:53:45,240 OR PRIVATE PRACTICE CAREER. 6639 04:53:45,240 --> 04:53:47,920 ON THE ACADEMIC SIDE, IN 6640 04:53:47,920 --> 04:53:49,080 GENERAL, IT'S NOT AS HIGHLY 6641 04:53:49,080 --> 04:53:51,400 THOUGHT OF WHEN YOU'RE GOING UP 6642 04:53:51,400 --> 04:53:53,560 FOR PROMOTION. 6643 04:53:53,560 --> 04:53:54,840 AS IT ALSO NOT AS EASY TO 6644 04:53:54,840 --> 04:54:01,040 SUPPORT AN ONGOING PROGRAM. 6645 04:54:01,040 --> 04:54:05,720 AND SO IT'S LESS ATTRACTIVE THAN 6646 04:54:05,720 --> 04:54:07,320 POTENTIALLY OTHER FIELDS. 6647 04:54:07,320 --> 04:54:09,560 THE MONETARY PART AND PEOPLE ARE 6648 04:54:09,560 --> 04:54:11,520 STARTING TO RECOGNIZE THIS IN 6649 04:54:11,520 --> 04:54:12,520 THE ALS ASSOCIATION JUST HAD A 6650 04:54:12,520 --> 04:54:15,360 GRANT FUNDING CALL TO FUND A 6651 04:54:15,360 --> 04:54:17,760 MEMBER OF CLINICAL TRIALS SITES 6652 04:54:17,760 --> 04:54:19,000 TO IMPROVE THEIR INFRASTRUCTURE 6653 04:54:19,000 --> 04:54:23,760 AND I THINK THAT'S A REALLY GOOD 6654 04:54:23,760 --> 04:54:27,120 EFFORT BUT I DON'T THINK IT 6655 04:54:27,120 --> 04:54:30,320 ADDRESS THE PANOPLY OF 6656 04:54:30,320 --> 04:54:33,040 CHALLENGES PEOPLE HAVE AND I 6657 04:54:33,040 --> 04:54:34,240 THINK CHIADI MENTIONED THE OTHER 6658 04:54:34,240 --> 04:54:35,920 IMPORTANT PART WHICH IS EVEN IT 6659 04:54:35,920 --> 04:54:40,720 THERE'S INFRASTRUCTURE AND 6660 04:54:40,720 --> 04:54:41,720 INCENTIVE, THERE IS A BOOK OF 6661 04:54:41,720 --> 04:54:43,440 KNOWLEDGE REQUIRED TO DO A GOOD 6662 04:54:43,440 --> 04:54:46,400 JOB ON THIS AND TO EVOLVE FROM 6663 04:54:46,400 --> 04:54:48,840 BEING A SITE INVESTIGATOR TO A 6664 04:54:48,840 --> 04:54:49,960 STUDY INVESTIGATOR TO DEVELOPING 6665 04:54:49,960 --> 04:54:53,080 YOUR OWN STUDIES. 6666 04:54:53,080 --> 04:54:55,160 I THINK WE DON'T HAVE ANYWHERE 6667 04:54:55,160 --> 04:54:55,720 NEAR THE INSTITUTIONAL 6668 04:54:55,720 --> 04:54:57,000 INFRASTRUCTURE TO DEVELOP THAT 6669 04:54:57,000 --> 04:55:02,160 AT THE PACE WE NEED. 6670 04:55:02,160 --> 04:55:05,480 >>WE HAVE A SHORTAGE. 6671 04:55:05,480 --> 04:55:08,880 WE TRAIN ALS TRIALISTS AND THEY 6672 04:55:08,880 --> 04:55:10,560 GET HIRED AWAY IMMEDIATELY FOR 6673 04:55:10,560 --> 04:55:12,960 INDUSTRY AND THAT'S GREAT FOR 6674 04:55:12,960 --> 04:55:16,360 INDUSTRY BUT WE DON'T HAVE THE 6675 04:55:16,360 --> 04:55:18,240 STAFFING TO ENROLL PEOPLE 6676 04:55:18,240 --> 04:55:19,280 EVERYDAY IN TRIALS AND GIVEN THE 6677 04:55:19,280 --> 04:55:21,760 NUMBER OF TRIALS OUT THERE. 6678 04:55:21,760 --> 04:55:24,200 I LOVE SOME OF THE IDEAS WE 6679 04:55:24,200 --> 04:55:28,680 HEARD FROM PANEL MEMBERS 6680 04:55:28,680 --> 04:55:33,760 INCREASE THE NUMBER OF CLINICAL 6681 04:55:33,760 --> 04:55:34,840 RESEARCHERS. 6682 04:55:34,840 --> 04:55:37,960 >>AND I WANT TO ASK THE 6683 04:55:37,960 --> 04:55:41,040 QUESTION FOR ANYONE. 6684 04:55:41,040 --> 04:55:43,880 CAN WE ENSURE EVERY CLINICAL 6685 04:55:43,880 --> 04:55:49,400 TRIAL SETS A TARGET ENROLLMENT 6686 04:55:49,400 --> 04:55:51,400 GOALS FOR PEOPLE OF COLOR AND 6687 04:55:51,400 --> 04:55:54,320 REPORT THEIR SUCCESS AT REACHING 6688 04:55:54,320 --> 04:55:56,960 THOSE GOALS TO THE COMMUNITY? 6689 04:55:56,960 --> 04:56:03,600 HOW CAN WE ASK FDA TO APPROVE 6690 04:56:03,600 --> 04:56:05,000 DRUGS BASED ON SUBSTANTIAL 6691 04:56:05,000 --> 04:56:10,000 EVIDENCE IF IT'S ONLY EVIDENCE 6692 04:56:10,000 --> 04:56:18,360 IN A WHITE POPULATION? 6693 04:56:18,360 --> 04:56:21,640 >>TIM, I KNOW YOU'RE INVOLVED 6694 04:56:21,640 --> 04:56:23,320 IN THE TAMBOURINE INITIATIVE. 6695 04:56:23,320 --> 04:56:28,560 >>I THINK IT'S RECOGNIZED THAT 6696 04:56:28,560 --> 04:56:29,440 ALS CLINICAL TRIALS AND CLINICS 6697 04:56:29,440 --> 04:56:32,000 AND WE'RE BEGINNING TO GATHER 6698 04:56:32,000 --> 04:56:33,760 DATA THROUGH THE NORTHEAST 6699 04:56:33,760 --> 04:56:39,600 CONSORTIUM FUNDED BY A GROUP 6700 04:56:39,600 --> 04:56:44,840 CALLED TAMBOURINE LOOK AT THE 6701 04:56:44,840 --> 04:56:46,840 MEDICARE DATABASES AND TABLE 1 6702 04:56:46,840 --> 04:56:47,640 OFTEN REPORTS THE INFORMATION 6703 04:56:47,640 --> 04:56:49,080 FOR CLINICAL TRIALS. 6704 04:56:49,080 --> 04:56:50,600 MY POINT IS IN CLINICAL TRIALS 6705 04:56:50,600 --> 04:56:55,080 AND IN CLINICS, THERE'S A LACK 6706 04:56:55,080 --> 04:56:56,480 OF REPRESENTATION OF DIFFERENT 6707 04:56:56,480 --> 04:56:59,720 ETHNIC GROUPS AND MINORITIES AND 6708 04:56:59,720 --> 04:57:03,680 PEOPLE IN RURAL POPULATIONS. 6709 04:57:03,680 --> 04:57:05,160 WHAT KIND OF DIFFERENCE WE STILL 6710 04:57:05,160 --> 04:57:07,840 WRAP OUR HEAD AROUND THAT BUT 6711 04:57:07,840 --> 04:57:08,600 IT'S STILL LOW. 6712 04:57:08,600 --> 04:57:09,440 I THINK IT IS A CHALLENGE IN 6713 04:57:09,440 --> 04:57:14,720 TERMS OF RECRUITING PEOPLE TO 6714 04:57:14,720 --> 04:57:16,560 CLINICS AND ALSO TO CLINICAL 6715 04:57:16,560 --> 04:57:18,840 TRIALS FOR A WHOLE VARIETY OF 6716 04:57:18,840 --> 04:57:20,160 REASONS THAT WE'RE JUST 6717 04:57:20,160 --> 04:57:20,880 BEGINNING TO WRAP OUR HEAD 6718 04:57:20,880 --> 04:57:23,760 AROUND AND IT'S IMPORTANT TO DO. 6719 04:57:23,760 --> 04:57:25,800 SO WE'RE TRYING TO PUSH THAT -- 6720 04:57:25,800 --> 04:57:27,720 WE DON'T HAVE ANY SOLUTIONS YET. 6721 04:57:27,720 --> 04:57:30,120 THAT'S STEP ONE TO ANSWERING THE 6722 04:57:30,120 --> 04:57:33,760 QUESTION ABOUT AND MAKING SURE 6723 04:57:33,760 --> 04:57:35,760 THE APPROVED DRUGS WORK IN ALL 6724 04:57:35,760 --> 04:57:40,680 POPULATIONS. 6725 04:57:40,680 --> 04:57:46,800 >>THAT'S A HUGE SETTING FOR 6726 04:57:46,800 --> 04:57:49,000 RECRUITING DIVERSITY AND MEETING 6727 04:57:49,000 --> 04:57:49,640 THE CRITERIA. 6728 04:57:49,640 --> 04:57:52,680 I DON'T MEAN THIS TO BE A PASS 6729 04:57:52,680 --> 04:57:54,800 THE BUCK ANSWER BUT IT'S AT 6730 04:57:54,800 --> 04:57:57,640 LEAST PARTIALLY A REFLECTION OF 6731 04:57:57,640 --> 04:57:58,520 OUR HEALTH CARE SYSTEM WHICH 6732 04:57:58,520 --> 04:58:00,360 DOESN'T PROVIDE THE SAME ACCESS 6733 04:58:00,360 --> 04:58:01,080 TO CARE ACROSS THE DIVERSE 6734 04:58:01,080 --> 04:58:06,240 COMMUNITY OF PEOPLE WHO LIVE IN 6735 04:58:06,240 --> 04:58:11,160 THE UNITED STATES. 6736 04:58:11,160 --> 04:58:13,120 IN ARIZONA WHERE I LIVE, THE 6737 04:58:13,120 --> 04:58:15,800 BIGGEST CHALLENGE IS 6738 04:58:15,800 --> 04:58:17,800 UNDOCUMENTED IMGRAMIGRANTS WHICS 6739 04:58:17,800 --> 04:58:19,440 A HUGE PART OF OUR POPULATION. 6740 04:58:19,440 --> 04:58:23,240 THERE'S FEAR THAT PREVENTS THEM 6741 04:58:23,240 --> 04:58:24,400 TO COME TO OUR CLINIC AND 6742 04:58:24,400 --> 04:58:25,520 RESOURCES THAT PREVENT THEM AND 6743 04:58:25,520 --> 04:58:27,760 I DON'T KNOW WE CAN COMPLETELY 6744 04:58:27,760 --> 04:58:29,880 SOLVE THIS BY IMPROVING THE 6745 04:58:29,880 --> 04:58:30,480 CLINICAL TRIAL INFRASTRUCTURE 6746 04:58:30,480 --> 04:58:39,560 THOUGH WE CERTAINLY SHOULD. 6747 04:58:39,560 --> 04:58:42,240 I THINK IT'S A BIGGER PROBLEM. 6748 04:58:42,240 --> 04:58:45,720 >>MANDATES HAVE NOT BEEN 6749 04:58:45,720 --> 04:58:46,000 SUCCESSFUL. 6750 04:58:46,000 --> 04:58:48,240 THAT'S WHY WE STILL TALK ABOUT 6751 04:58:48,240 --> 04:58:49,480 THIS PROBLEM. 6752 04:58:49,480 --> 04:58:49,960 ESSENTIALLY WHAT THE 6753 04:58:49,960 --> 04:58:56,040 CONVERSATION IS ABOUT IS THE 6754 04:58:56,040 --> 04:59:01,480 MAKING THE RESEARCH EERS 6755 04:59:01,480 --> 04:59:04,000 DEMONSTRATE INCLUSIVENESS OR 6756 04:59:04,000 --> 04:59:06,400 ELSE AND WHAT WORKS IS NOTE 6757 04:59:06,400 --> 04:59:07,480 SETTING MANDATES FOR THE 6758 04:59:07,480 --> 04:59:08,080 INVESTIGATORS BUT SUPPORTING 6759 04:59:08,080 --> 04:59:10,560 COMMUNITY ENGAGEMENT. 6760 04:59:10,560 --> 04:59:11,800 IT'S COMMUNITY ENGAGEMENT HAS 6761 04:59:11,800 --> 04:59:13,760 BEEN SHOWN OVER AND OVER AGAIN 6762 04:59:13,760 --> 04:59:15,960 THE WAY TO DRIVE PARTICIPATION 6763 04:59:15,960 --> 04:59:18,320 IN DISADVANTAGED COMMUNITIES. 6764 04:59:18,320 --> 04:59:23,400 WE OUGHT TO HAVE A REORIENTATION 6765 04:59:23,400 --> 04:59:26,600 TO DEMANDING MANDATES TO 6766 04:59:26,600 --> 04:59:29,680 IMPORTING AND INCENTIVIZING 6767 04:59:29,680 --> 04:59:31,120 COMMUNITY-ENGAGEMENT EFFORTS. 6768 04:59:31,120 --> 04:59:33,640 I THINK WE'LL GETTING BETTER 6769 04:59:33,640 --> 04:59:35,400 RESULTS IF WE ENCOURAGE 6770 04:59:35,400 --> 04:59:37,800 COMMUNITY ENGAGEMENT AND THE 6771 04:59:37,800 --> 04:59:39,840 OTHER ADVANTAGE IS IT PROVIDES 6772 04:59:39,840 --> 04:59:41,440 OPPORTUNITIES FOR THE COMMUNITY 6773 04:59:41,440 --> 04:59:45,400 TO HAVE SOME OWNERSHIP IN THE 6774 04:59:45,400 --> 04:59:46,720 RESEARCH AND BE ABLE TO DRIVER 6775 04:59:46,720 --> 04:59:47,760 SOME RESEARCH QUESTIONS 6776 04:59:47,760 --> 04:59:51,080 OTHERWISE YOU HAVE AN 6777 04:59:51,080 --> 04:59:53,000 INVESTIGATOR THAT'S COME UP WITH 6778 04:59:53,000 --> 04:59:55,920 AN IDEA IN THEIR WEB A.R. AND 6779 04:59:55,920 --> 04:59:57,480 COMING TO A COMMUNITY AND SAYING 6780 04:59:57,480 --> 04:59:58,800 COME DO WHAT I WANT TO YOU DO IN 6781 04:59:58,800 --> 04:59:59,920 THE RESEARCH AND YOU WANT A 6782 04:59:59,920 --> 05:00:01,360 PROJECT THAT RESONATES WITH THE 6783 05:00:01,360 --> 05:00:02,520 COMMUNITY BECAUSE IT'S ANSWERING 6784 05:00:02,520 --> 05:00:03,800 QUESTIONS THAT THEY WOULD LIKE 6785 05:00:03,800 --> 05:00:08,200 TO HAVE ANSWERED AND YOU ONLY 6786 05:00:08,200 --> 05:00:09,720 KNOW THE QUESTIONS THEY WOULD 6787 05:00:09,720 --> 05:00:12,360 LIKE TO HAVE ANSWERED IF YOU ARE 6788 05:00:12,360 --> 05:00:13,480 ENGAGED IN A CONVERSATION. 6789 05:00:13,480 --> 05:00:14,560 I THINK COMMUNITY ENGAGEMENT IS 6790 05:00:14,560 --> 05:00:21,920 CRUCIAL. 6791 05:00:21,920 --> 05:00:23,160 IT SHOULD BE AN INVESTMENT. 6792 05:00:23,160 --> 05:00:33,600 >>SO WELL SAID, CHIADI. 6793 05:00:33,840 --> 05:00:35,280 >>DIAGNOSTIC DELAY IS WORSE IN 6794 05:00:35,280 --> 05:00:36,200 PEOPLE OF COLOR. 6795 05:00:36,200 --> 05:00:38,440 I THINK YOU HAVE TO TAKE IT A 6796 05:00:38,440 --> 05:00:40,360 STEP BACK THAT THAT IS GOING TO 6797 05:00:40,360 --> 05:00:41,760 TRICKLE DOWN INTO TRIALS AND WHO 6798 05:00:41,760 --> 05:00:52,320 IS ELIGIBLE AND I THINK WE NEED 6799 05:00:54,720 --> 05:01:00,360 TO TAKE A FIRST STEP IN 6800 05:01:00,360 --> 05:01:00,960 ADDRESSING THIS. 6801 05:01:00,960 --> 05:01:04,640 >>I WANT TO SEE IF PANELISTS 6802 05:01:04,640 --> 05:01:09,320 SEE A ROLE FOR NON-TRADITIONAL 6803 05:01:09,320 --> 05:01:11,120 SITES TO BRING CLINICAL 6804 05:01:11,120 --> 05:01:12,840 PARTICIPATION TO A LARGER 6805 05:01:12,840 --> 05:01:13,760 PERCENTAGE OF THE POPULATION 6806 05:01:13,760 --> 05:01:17,720 PERHAPS WITH EXPANDED ACCESS OR 6807 05:01:17,720 --> 05:01:21,760 A BIOMARKER OR A STUDY WITH 6808 05:01:21,760 --> 05:01:24,240 LOWER BURDEN. 6809 05:01:24,240 --> 05:01:27,040 >>ONE MINUTE REMAINING. 6810 05:01:27,040 --> 05:01:28,400 I'LL OPEN IT TO ANYONE IN THE 6811 05:01:28,400 --> 05:01:28,600 PANEL. 6812 05:01:28,600 --> 05:01:36,480 >>YES. 6813 05:01:36,480 --> 05:01:39,400 >>YES, AND WE SHOULD THINK OF A 6814 05:01:39,400 --> 05:01:40,360 TOOLBOX TO DEVELOP TO HELP 6815 05:01:40,360 --> 05:01:51,040 ENGAGE AND MAKE IT EASIER TO DO 6816 05:01:51,040 --> 05:01:51,680 STARTING FROM SCRATCH IS 6817 05:01:51,680 --> 05:01:54,320 DIFFICULT FOR A HARD-RESOURCED 6818 05:01:54,320 --> 05:01:56,920 AREA AND WE NEED A TOOL TO MAKE 6819 05:01:56,920 --> 05:01:59,720 IT EASIER TO JUMP IN. 6820 05:01:59,720 --> 05:02:02,840 >>AND NOT BECOME SOMETHING THAT 6821 05:02:02,840 --> 05:02:03,800 COMPETES WITH THE FIRST PRIORITY 6822 05:02:03,800 --> 05:02:09,800 WHICH IS TO PROVIDE THE CARE. 6823 05:02:09,800 --> 05:02:17,800 >>AGREED. 6824 05:02:17,800 --> 05:02:25,800 >>MAYBE TERTIARY SITES CAN 6825 05:02:25,800 --> 05:02:32,400 UTILIZE MONITORING TO SUPPORT 6826 05:02:32,400 --> 05:02:32,880 SITES. 6827 05:02:32,880 --> 05:02:36,320 >>I THINK THIS WILL CONCLUDE 6828 05:02:36,320 --> 05:02:36,920 THE CLINICAL RESEARCH PANEL. 6829 05:02:36,920 --> 05:02:40,040 THANK YOU. 6830 05:02:40,040 --> 05:02:41,760 >>THANK YOU. 6831 05:02:41,760 --> 05:02:42,200 THANKS TO EVERYONE. 6832 05:02:42,200 --> 05:02:46,320 THANKS FOR THE QUESTION. 6833 05:02:46,320 --> 05:02:47,400 >>THANKS FOR THE EXCELLENT 6834 05:02:47,400 --> 05:02:47,680 MODERATION. 6835 05:02:47,680 --> 05:02:52,000 >>YOU'RE WELCOME. 6836 05:02:52,000 --> 05:02:53,360 >>THANK YOU VERY MUCH. 6837 05:02:53,360 --> 05:02:54,960 THAT WAS AN EXCELLENT SESSION 6838 05:02:54,960 --> 05:02:56,440 AND WONDERFUL TO SEE THAT MUCH 6839 05:02:56,440 --> 05:02:57,480 GIVE AND TAKE. 6840 05:02:57,480 --> 05:02:58,840 GREAT NUMBER OF GOOD QUESTIONS. 6841 05:02:58,840 --> 05:03:05,320 THANK YOU FOR THAT. 6842 05:03:05,320 --> 05:03:07,040 WE'LL GO TO THE CONCLUDING 6843 05:03:07,040 --> 05:03:09,760 ASPECT OF WHICH IS A SERIES OF 6844 05:03:09,760 --> 05:03:13,760 COMMENTARIES THAT HAVE BEEN 6845 05:03:13,760 --> 05:03:15,040 SUBMITTED. 6846 05:03:15,040 --> 05:03:16,320 THE PROCEDURE HERE IS THAT THESE 6847 05:03:16,320 --> 05:03:17,800 ARE OPEN FOR PUBLIC COMMENT AND 6848 05:03:17,800 --> 05:03:19,480 IF YOU WANT TO MAKE A COMMENT, 6849 05:03:19,480 --> 05:03:21,800 PLEASE TYPE IT IN THE QUESTION 6850 05:03:21,800 --> 05:03:22,880 AND ANSWER BOX. 6851 05:03:22,880 --> 05:03:27,720 FIRST, TYPE ALLOWED IF YOU WOULD 6852 05:03:27,720 --> 05:03:29,080 LIKE TO READ YOUR COMMENT 6853 05:03:29,080 --> 05:03:31,000 YOURSELF OR READ IF YOU'D LIKE 6854 05:03:31,000 --> 05:03:31,760 IT READ FOR YOU. 6855 05:03:31,760 --> 05:03:33,440 WE'LL IDENTIFY YOU WITH YOUR 6856 05:03:33,440 --> 05:03:35,520 NAME IN ZOOM UNLESS YOU ADD 6857 05:03:35,520 --> 05:03:35,760 ANONYMOUS. 6858 05:03:35,760 --> 05:03:38,560 THEN WE WILL HAVE YOU TYPE THE 6859 05:03:38,560 --> 05:03:39,920 REST OF THE COMMENT AND WE'LL 6860 05:03:39,920 --> 05:03:40,560 READ COMMENTS THAT WERE SHARED 6861 05:03:40,560 --> 05:03:49,280 IN ADVANCE. 6862 05:03:49,280 --> 05:03:51,400 THE FIRST COMMENT SAID THOSE OF 6863 05:03:51,400 --> 05:03:55,040 US BATTLING ALS ARE LOOKING FOR 6864 05:03:55,040 --> 05:03:56,120 RESOLUTIONS WITH LIFE 6865 05:03:56,120 --> 05:03:57,200 EXPECTATIONS SHORT TWO YEARS 6866 05:03:57,200 --> 05:03:59,880 EVERY MINUTE IS APPRECIATION 6867 05:03:59,880 --> 05:04:02,200 AND -- PRECIOUS AND WE DON'T 6868 05:04:02,200 --> 05:04:04,640 HAVE THE LUXURY OF TIME FOR 6869 05:04:04,640 --> 05:04:06,080 SOLUTIONS BUT HOPE YOU SOLVE 6870 05:04:06,080 --> 05:04:08,080 THIS PLAGUE RIGHT NOW IF NOT IN 6871 05:04:08,080 --> 05:04:11,440 MY LIMITED LIFE TIME AT LEAST IN 6872 05:04:11,440 --> 05:04:12,760 THE NEAR TERM. 6873 05:04:12,760 --> 05:04:23,480 THANK YOU AND SEMPER FIDELIS. 6874 05:04:23,480 --> 05:04:27,600 >>I HAVE AN ANONYMOUS QUESTION 6875 05:04:27,600 --> 05:04:31,920 FOR ALS CARRIERS IS OF THE 6876 05:04:31,920 --> 05:04:36,560 UTMOST IMPORTANCE AS IS COULD 6877 05:04:36,560 --> 05:04:40,040 GREATLY DELAY HALT DISEASE 6878 05:04:40,040 --> 05:04:40,640 PROGRESSION. 6879 05:04:40,640 --> 05:04:42,680 WE HAVE FAMILIAL ALS IN OUR 6880 05:04:42,680 --> 05:04:44,440 FAMILY AND WATCHING IT RIP 6881 05:04:44,440 --> 05:04:46,720 THROUGH OUR LOVED ONES IS HARDER 6882 05:04:46,720 --> 05:04:48,800 THAN ANYONE CAN IMAGINE. 6883 05:04:48,800 --> 05:04:51,960 USE THE TREATMENT OPTIONS BEFORE 6884 05:04:51,960 --> 05:04:57,000 THE DISEASE STARTS AND GENE AREA 6885 05:04:57,000 --> 05:04:57,800 CARRIERS. 6886 05:04:57,800 --> 05:05:01,200 THIS IDEA MUST BE CONSIDERED. 6887 05:05:01,200 --> 05:05:02,680 IF THE ONLY OPTION IS TO DO 6888 05:05:02,680 --> 05:05:04,360 NOTHING, WHAT WOULD YOU CHOOSE? 6889 05:05:04,360 --> 05:05:12,800 THANK YOU. 6890 05:05:12,800 --> 05:05:16,400 >>OUR NEXT COMMENT IS FROM 6891 05:05:16,400 --> 05:05:18,320 CASSANDRA HADAD. 6892 05:05:18,320 --> 05:05:19,880 >>THANK YOU. 6893 05:05:19,880 --> 05:05:22,160 I WANT TO START BY THANK YOU ALL 6894 05:05:22,160 --> 05:05:23,320 FOR YOUR HARD WORK AND 6895 05:05:23,320 --> 05:05:27,560 DEDICATION TO THE ALS COMMUNITY. 6896 05:05:27,560 --> 05:05:32,640 AS I READ THROUGH THE STRATEGIC 6897 05:05:32,640 --> 05:05:34,760 PLAN I SEE YOUR HOPE SHOWING 6898 05:05:34,760 --> 05:05:36,280 THROUGH AND THANK YOU FOR 6899 05:05:36,280 --> 05:05:36,920 INTEGRATING THE COMMUNITY AND 6900 05:05:36,920 --> 05:05:38,000 WHY PEOPLE SPEAKING TO YOU 6901 05:05:38,000 --> 05:05:38,440 TODAY. 6902 05:05:38,440 --> 05:05:41,240 AUGUST 30 OF THIS YEAR MY MOTHER 6903 05:05:41,240 --> 05:05:42,280 DIED OF ALS. 6904 05:05:42,280 --> 05:05:44,320 SHE WAS AT LEAST THE 33rd PERSON 6905 05:05:44,320 --> 05:05:47,080 IN OUR FAMILY. 6906 05:05:47,080 --> 05:05:50,240 WE HAVE RAPIDLY AGGRESSIVE 6907 05:05:50,240 --> 05:05:52,200 SOD1A5B SO OUR FAMILY DIES 6908 05:05:52,200 --> 05:05:52,520 QUICKLY. 6909 05:05:52,520 --> 05:05:53,760 BECAUSE OF ADVOCACY AND TRIALS 6910 05:05:53,760 --> 05:05:57,000 AND EXPANDED ACCESS, MY MOTHER 6911 05:05:57,000 --> 05:05:58,160 LIVED 25 MONTHS FROM SYSTEM 6912 05:05:58,160 --> 05:05:58,360 ONSET. 6913 05:05:58,360 --> 05:06:03,440 THE LONGEST IN OUR FAMILY. 6914 05:06:03,440 --> 05:06:05,080 THOSE EXTRA FEW MONTHS MEANT 6915 05:06:05,080 --> 05:06:07,760 EVERYTHING TO MY FAMILY AND WITH 6916 05:06:07,760 --> 05:06:10,760 YOUNG CHILDREN IT GAVE MOM MORE 6917 05:06:10,760 --> 05:06:12,840 OPPORTUNITIES TO MAKE MEMORIES 6918 05:06:12,840 --> 05:06:13,800 BECAUSE OF RESEARCH AND ADVOCACY 6919 05:06:13,800 --> 05:06:14,840 BECAUSE OF PEOPLE LIKE EVERYONE 6920 05:06:14,840 --> 05:06:15,520 HERE TODAY. 6921 05:06:15,520 --> 05:06:16,760 THE ONLY REASON SHE GOT INTO 6922 05:06:16,760 --> 05:06:20,680 TRIALS AND EXPANDED ACCESS WAS 6923 05:06:20,680 --> 05:06:22,600 THROUGH MY FAMILY FIGHTING EVERY 6924 05:06:22,600 --> 05:06:25,320 STEP OF THE WAY FOR HER AND 6925 05:06:25,320 --> 05:06:29,120 DR. QUIN A TRUE ALS HERO GETTING 6926 05:06:29,120 --> 05:06:29,960 MEDICATION FOR MY MOM. 6927 05:06:29,960 --> 05:06:31,840 THERE NEEDS TO BE MORE RESOURCES 6928 05:06:31,840 --> 05:06:33,760 TO GUIDE PATIENTS THROUGH THIS 6929 05:06:33,760 --> 05:06:36,920 PART OF THE JOURNEY. 6930 05:06:36,920 --> 05:06:38,800 THANK YOU FOR RECOGNIZING THE 6931 05:06:38,800 --> 05:06:40,800 SIGNIFICANT MENTAL AND FINANCIAL 6932 05:06:40,800 --> 05:06:42,480 IMPACT CARING FOR A LOVED ONE 6933 05:06:42,480 --> 05:06:44,440 WITH ALS BRINGS AND THERE'S A 6934 05:06:44,440 --> 05:06:47,760 SIGNIFICANT FINANCIAL IMPACT FOR 6935 05:06:47,760 --> 05:06:50,080 THOSE PARTICIPATING IN GENETIC 6936 05:06:50,080 --> 05:06:51,800 RESEARCH AS CARRIERS. 6937 05:06:51,800 --> 05:06:53,040 THIS MUST BE ADDRESSED. 6938 05:06:53,040 --> 05:06:56,400 I KNOW IF I MY GENE TURNS ON AND 6939 05:06:56,400 --> 05:06:59,000 GET DIAGNOSED WITH ALS I WILL 6940 05:06:59,000 --> 05:07:00,800 DIE BECAUSE OF NOW THERE'S NO 6941 05:07:00,800 --> 05:07:01,000 HOPE. 6942 05:07:01,000 --> 05:07:10,400 IF I'M LUCKY I MAY GET TWO EXTRA 6943 05:07:10,400 --> 05:07:16,440 YEARS WITH MY TWINS AND I 6944 05:07:16,440 --> 05:07:18,040 IMPLORE YOU TO ADD AN AGGRESSIVE 6945 05:07:18,040 --> 05:07:19,880 TIME LINE TO THE STRATEGIC PLAN. 6946 05:07:19,880 --> 05:07:21,800 IT'S PACKED FULL OF GREAT IDEAS 6947 05:07:21,800 --> 05:07:25,800 AND WONDERFUL INTENTIONS BUT THE 6948 05:07:25,800 --> 05:07:30,240 RIGHT TRAJECTORY BUT MYSELF AND 6949 05:07:30,240 --> 05:07:33,800 MY CHILDREN ARE COUNTING ON A 6950 05:07:33,800 --> 05:07:34,000 CURE. 6951 05:07:34,000 --> 05:07:38,760 THE CLOCK IS TICKING AND MUST 6952 05:07:38,760 --> 05:07:41,760 MOVE FORWARD TO INTERVENE AND 6953 05:07:41,760 --> 05:07:45,400 IDENTIFY WHEN TO ACT IN THE 6954 05:07:45,400 --> 05:07:47,400 ASYMPTOMATIC POPULATION TO 6955 05:07:47,400 --> 05:07:49,360 PREVENT ALS AND LOOK AT THE 6956 05:07:49,360 --> 05:07:50,960 AMOUNT PEOPLE ARE WILLING TO 6957 05:07:50,960 --> 05:07:53,360 TAKE WITH THE GEN PENETRANTS 6958 05:07:53,360 --> 05:07:56,040 RATE OF MORE THAN 90% AND 6959 05:07:56,040 --> 05:07:57,800 RESEARCH THAT CAN CHANGE MY 6960 05:07:57,800 --> 05:07:59,040 CHILDREN'S LIVES I WILL 6961 05:07:59,040 --> 05:08:00,880 LITERALLY TRY EVERYTHING. 6962 05:08:00,880 --> 05:08:02,720 I'M INVOLVED IN COLUMBIA AND 6963 05:08:02,720 --> 05:08:04,880 MASS GENERAL'S OBSERVATIONAL 6964 05:08:04,880 --> 05:08:07,240 TRIALS AND BIOGEN'S ATLAS. 6965 05:08:07,240 --> 05:08:13,680 I'M DOING EVERYTHING ON MY PART 6966 05:08:13,680 --> 05:08:17,720 TO HELP BUT DOESN'T SEEM LIKE 6967 05:08:17,720 --> 05:08:17,960 ENOUGH. 6968 05:08:17,960 --> 05:08:19,000 THE COMMUNITY IS WILLING TO 6969 05:08:19,000 --> 05:08:20,240 SERVE AS BOOTS ON THE GROUND. 6970 05:08:20,240 --> 05:08:21,600 THANK YOU FOR TAKING THE TIME TO 6971 05:08:21,600 --> 05:08:23,200 HEAR MY COMMENTS. 6972 05:08:23,200 --> 05:08:24,640 IF YOU REMEMBER ONE THING ABOUT 6973 05:08:24,640 --> 05:08:27,120 ME AND MY ALS FAMILY REMEMBER WE 6974 05:08:27,120 --> 05:08:28,280 NEED TO YOU FIND THE OPTIMUM 6975 05:08:28,280 --> 05:08:29,480 TIME TO INTERVENE BEFORE IT'S 6976 05:08:29,480 --> 05:08:30,320 TOO LATE. 6977 05:08:30,320 --> 05:08:40,880 I KNOW TOGETHER WE CAN END ALS. 6978 05:08:53,840 --> 05:08:58,080 >>I AM MINDY ERRLAB BY A GROUP 6979 05:08:58,080 --> 05:09:00,320 OF VOLUNTEERS IMPACTED BY 6980 05:09:00,320 --> 05:09:03,280 GENETIC ALS AND GENETIC FTD. 6981 05:09:03,280 --> 05:09:06,760 WE WANT TO THANK THE NIH FOR 6982 05:09:06,760 --> 05:09:10,080 RECOGNIZING THE HUNDREDS OF 6983 05:09:10,080 --> 05:09:15,320 AMERICANS AT RISK AS STAKEH 6984 05:09:15,320 --> 05:09:16,960 STAKEHOLDERS AND THANK THE 6985 05:09:16,960 --> 05:09:20,080 MEMBERS WHO CREATED THE 6986 05:09:20,080 --> 05:09:20,360 DOCUMENT. 6987 05:09:20,360 --> 05:09:21,280 WE'RE PLEASED OUR COMMUNITY IS 6988 05:09:21,280 --> 05:09:22,920 CONSIDERED A PRIORITY. 6989 05:09:22,920 --> 05:09:25,280 WE'RE GRATEFUL THE COMMON SENSE 6990 05:09:25,280 --> 05:09:26,600 CALL TO INVESTIGATE POTENTIAL 6991 05:09:26,600 --> 05:09:27,320 THERAPIES AT ALL STAGES OF 6992 05:09:27,320 --> 05:09:32,080 DISEASE WAS INCLUDED. 6993 05:09:32,080 --> 05:09:37,400 WE WANTED TO POINT OUT ONE 6994 05:09:37,400 --> 05:09:37,800 WEAKNESS. 6995 05:09:37,800 --> 05:09:39,680 ALS HAS THREE MODIFYING DRUGS 6996 05:09:39,680 --> 05:09:44,120 THE FIRST APPROVED IN 1995. 6997 05:09:44,120 --> 05:09:46,560 IT'S INCOMPREHENSIBLE WHEN TO 6998 05:09:46,560 --> 05:09:48,280 INITIATE THE THERAPIES HAS NEVER 6999 05:09:48,280 --> 05:09:49,760 BEEN ASKED. 7000 05:09:49,760 --> 05:09:51,680 HOW MANY MORE THERAPIES FOR ALS 7001 05:09:51,680 --> 05:09:53,120 WILL BE APPROVED WITH NO 7002 05:09:53,120 --> 05:09:54,760 INVESTIGATION INTO WHETHER THEY 7003 05:09:54,760 --> 05:09:57,080 CAN FORESTALL STABILITY AND 7004 05:09:57,080 --> 05:09:58,440 DEATH MORE EFFECTIVELY WITH 7005 05:09:58,440 --> 05:10:02,640 EARLIER ADMINISTRATION? 7006 05:10:02,640 --> 05:10:04,640 OUR PRE-SYMPTOMATIC COMMUNITY'S 7007 05:10:04,640 --> 05:10:05,000 HEALTH MATTERS. 7008 05:10:05,000 --> 05:10:05,440 THANK YOU FOR YOUR 7009 05:10:05,440 --> 05:10:15,680 CONSIDERATION. 7010 05:10:25,280 --> 05:10:27,640 >>ARE YOU GOING TO JOIN US, 7011 05:10:27,640 --> 05:10:29,800 ELLEN? 7012 05:10:29,800 --> 05:10:36,840 >>I'M HERE. 7013 05:10:36,840 --> 05:10:38,840 I'VE UNMUTED. 7014 05:10:38,840 --> 05:10:40,280 KAREN ROBERTS STATES LOTS OF 7015 05:10:40,280 --> 05:10:42,840 TREATMENT IN DEVELOPMENT COULD 7016 05:10:42,840 --> 05:10:44,080 BE TRIED EARLIER TO PREVENT THE 7017 05:10:44,080 --> 05:10:47,760 DISEASE EVER STARTING. 7018 05:10:47,760 --> 05:10:50,200 SEEMS LUDICROUS TO ONLY FOCUS ON 7019 05:10:50,200 --> 05:10:51,600 TREATMENTS AFTER SYMPTOMS HAVE 7020 05:10:51,600 --> 05:10:51,840 STARTED. 7021 05:10:51,840 --> 05:10:55,520 PLEASE FOCUS ON PREVENTING ALS 7022 05:10:55,520 --> 05:10:57,760 FROM THE DEVELOPING AN AT-RISK 7023 05:10:57,760 --> 05:10:58,960 GENE CARRIERS. 7024 05:10:58,960 --> 05:11:00,040 PLEASE RESEARCH PREVENTIVE 7025 05:11:00,040 --> 05:11:01,840 TREATMENTS FOR THOSE WITH 7026 05:11:01,840 --> 05:11:05,800 RELEVANT GENE DEFECTS WHO ARE 7027 05:11:05,800 --> 05:11:09,240 NOT SHOWING DISEASE. 7028 05:11:09,240 --> 05:11:10,640 >>THE NEXT COMMENT IS ANONYMOUS 7029 05:11:10,640 --> 05:11:13,800 BUT FOLLOWS FROM THE PREVIOUS 7030 05:11:13,800 --> 05:11:16,080 CLINICAL TRIALS ARE NEEDED FOR 7031 05:11:16,080 --> 05:11:17,760 PRY-SYMPTOMATIC GENE CARRIERS TO 7032 05:11:17,760 --> 05:11:23,320 FOCUS ON PREVENTION. 7033 05:11:23,320 --> 05:11:24,720 >>THE NEXT COMMENT IS FROM 7034 05:11:24,720 --> 05:11:25,760 NEIL. 7035 05:11:25,760 --> 05:11:36,200 POLICE UNMUTE YOURSELF. 7036 05:11:53,880 --> 05:11:57,120 AND WE NEED TO FOCUS ON TIME TO 7037 05:11:57,120 --> 05:11:58,680 HUMAN IMPACT, TIME TO REAL 7038 05:11:58,680 --> 05:12:00,600 CHANGE IN THE LIVES OF PEOPLE 7039 05:12:00,600 --> 05:12:03,080 WITH ALS AND CONSIDER THE 7040 05:12:03,080 --> 05:12:04,400 SPECIFIC MILESTONE TO GET US 7041 05:12:04,400 --> 05:12:04,960 THERE. 7042 05:12:04,960 --> 05:12:06,440 I DIDN'T SEE TIME LINES IN THE 7043 05:12:06,440 --> 05:12:10,200 PLAN AND I THINK IT NEEDS IT. 7044 05:12:10,200 --> 05:12:12,280 I WOULD ALSO LIKE TO YOU THINK 7045 05:12:12,280 --> 05:12:13,840 OF THE BALANCE OF RESEARCH AND 7046 05:12:13,840 --> 05:12:15,000 INVESTMENTS ACROSS THE PLAN IN 7047 05:12:15,000 --> 05:12:17,680 TERMS OF TIME AND RESOURCES. 7048 05:12:17,680 --> 05:12:20,080 THESE RESEARCH EFFORTS ARE 7049 05:12:20,080 --> 05:12:21,200 WORKING ON DIFFERENT TIME 7050 05:12:21,200 --> 05:12:21,680 SCALES. 7051 05:12:21,680 --> 05:12:22,480 PLEASE BE SPECIFIC ABOUT WHAT 7052 05:12:22,480 --> 05:12:23,840 THE TIME SCALES ARE SO THE 7053 05:12:23,840 --> 05:12:25,240 COMMUNITY CAN MORE FULLY 7054 05:12:25,240 --> 05:12:25,800 UNDERSTAND THE PLAN AND ITS 7055 05:12:25,800 --> 05:12:29,360 POTENTIAL. 7056 05:12:29,360 --> 05:12:30,520 OUR EXISTING RESEARCH CAPACITY 7057 05:12:30,520 --> 05:12:33,160 ARE PEOPLE AND INSTITUTIONS ARE 7058 05:12:33,160 --> 05:12:34,080 ALSO NOT EVENLY DISTRIBUTED 7059 05:12:34,080 --> 05:12:36,720 AROUND THE COUNTRY. 7060 05:12:36,720 --> 05:12:38,160 WITHOUT CAREFUL PLANNING WE'LL 7061 05:12:38,160 --> 05:12:41,120 NOT GIVE THE RESEARCH AND 7062 05:12:41,120 --> 05:12:42,560 RESULTS WE NEED WE'LL END UP IN 7063 05:12:42,560 --> 05:12:45,200 THE SAME PLACE WE ARE TODAY AND 7064 05:12:45,200 --> 05:12:46,160 JUST GET MORE APPLICATIONS FROM 7065 05:12:46,160 --> 05:12:47,280 THE STATEMENT PLACES AND 7066 05:12:47,280 --> 05:12:49,280 SPECIFICALLY AND IMPORTANTLY WE 7067 05:12:49,280 --> 05:12:51,560 NEED TO CONSIDER TRIAL CAPACITY. 7068 05:12:51,560 --> 05:12:53,480 THERE'S AN EMPHASIS IN THE DRAFT 7069 05:12:53,480 --> 05:12:55,880 AND PERHAPS A RISK TO FURTHER 7070 05:12:55,880 --> 05:12:57,640 CENTRALIZE RESEARCH CAPACITY AND 7071 05:12:57,640 --> 05:12:58,600 THAT MIGHT BE THE RIGHT STRATEGY 7072 05:12:58,600 --> 05:13:00,240 FOR FUTURE OF WORK AND FOR HUMAN 7073 05:13:00,240 --> 05:13:03,120 RESEARCH IT MIGHT BE COUNTER 7074 05:13:03,120 --> 05:13:03,480 PRODUCTIVE. 7075 05:13:03,480 --> 05:13:04,920 WE NEED TO RUN CLINICAL TRIALS 7076 05:13:04,920 --> 05:13:06,200 WHERE PEOPLE WITH ALS LIVE NOT 7077 05:13:06,200 --> 05:13:07,520 WHERE ALS SCIENTISTS ARE 7078 05:13:07,520 --> 05:13:08,080 CURRENTLY LIVING. 7079 05:13:08,080 --> 05:13:09,760 SO WE NEED TO WORK ON THAT 7080 05:13:09,760 --> 05:13:19,920 PROBLEM. 7081 05:13:33,040 --> 05:13:37,720 AND WE NEED TO JUST GET 7082 05:13:37,720 --> 05:13:39,320 TREATMENTS AND CURES INTO PUB 7083 05:13:39,320 --> 05:13:39,520 MED. 7084 05:13:39,520 --> 05:13:41,880 >>THANK YOU, NEIL. 7085 05:13:41,880 --> 05:13:43,160 OUR NEXT COMMENT IS FROM MIKE. 7086 05:13:43,160 --> 05:13:51,640 PLEASE UNMUTE YOURSELF. 7087 05:13:51,640 --> 05:13:52,440 >>THANK YOU. 7088 05:13:52,440 --> 05:13:57,160 I WANT TO APPLAUD THE GROUP ON A 7089 05:13:57,160 --> 05:14:01,560 VERY BROAD AND WELL THOUGHT. 7090 05:14:01,560 --> 05:14:04,360 OUT STRATEGY OF RESEARCH MY 7091 05:14:04,360 --> 05:14:05,760 CONCERN IS IS IT BEING 7092 05:14:05,760 --> 05:14:07,640 ACTIONABLE AND NOT BECAUSE THE 7093 05:14:07,640 --> 05:14:12,920 RESEARCH ISN'T SOLID AND 7094 05:14:12,920 --> 05:14:15,240 ACHIEVABLE BUT TOO MUCH AND I 7095 05:14:15,240 --> 05:14:16,080 FIND IT TOO BROAD. 7096 05:14:16,080 --> 05:14:18,720 I FEEL WE NEED TO TURN THE 7097 05:14:18,720 --> 05:14:19,960 STRATEGY INTO A PLAN AND NEEDS 7098 05:14:19,960 --> 05:14:30,520 TO HAVE DISCOVERY MILESTONES AND 7099 05:14:35,400 --> 05:14:36,880 RATHER THAN TO ATTACK THE 7100 05:14:36,880 --> 05:14:38,600 DISEASE MECHANISM, ETCETERA. 7101 05:14:38,600 --> 05:14:38,960 ALL AT ONE TIME. 7102 05:14:38,960 --> 05:14:43,960 THANK YOU. 7103 05:14:43,960 --> 05:14:44,680 >>THANK YOU, MIKE. 7104 05:14:44,680 --> 05:14:46,080 NOW BACK TO RITA. 7105 05:14:46,080 --> 05:14:51,960 >>I'M GOING READ A COMMENT. 7106 05:14:51,960 --> 05:14:54,440 I'M A C9 CARRIER AND DIAGNOSED 7107 05:14:54,440 --> 05:14:55,440 IN APRIL OF THIS YEAR. 7108 05:14:55,440 --> 05:14:57,440 I DETECTED DIFFICULTIES WITH MY 7109 05:14:57,440 --> 05:14:59,640 SPEECH STARTLING IN DECEMBER 7110 05:14:59,640 --> 05:15:00,640 2021 SO CONSIDERED MYSELF 7111 05:15:00,640 --> 05:15:01,840 FORTUNATE TO BE TREATED SO SOON 7112 05:15:01,840 --> 05:15:05,840 AFTER MY SYMPTOMS BEGAN. 7113 05:15:05,840 --> 05:15:11,240 HOWEVER MUCH TO MY DISMAY MY 7114 05:15:11,240 --> 05:15:13,760 NEUROLOGIST INDICATED THE 7115 05:15:13,760 --> 05:15:18,480 SYMPTOMS STARTED A YEAR PRIOR 7116 05:15:18,480 --> 05:15:18,920 DETECTING ANYTHING. 7117 05:15:18,920 --> 05:15:20,840 I THINK I COULD HAVE STARTED 7118 05:15:20,840 --> 05:15:21,280 TREATMENT EARLIER. 7119 05:15:21,280 --> 05:15:23,080 TO THINK I WOULD HAVE HAD MORE 7120 05:15:23,080 --> 05:15:24,960 FUNCTION NOW THAN 10 MONTHS INTO 7121 05:15:24,960 --> 05:15:26,760 THE DISEASE. 7122 05:15:26,760 --> 05:15:28,360 RECENTLY RESEARCH HAS BEEN 7123 05:15:28,360 --> 05:15:29,480 PUBLISHED DOCUMENTING THE 7124 05:15:29,480 --> 05:15:33,480 PRESENCE OF TD43 A HALLMARK OF 7125 05:15:33,480 --> 05:15:37,000 ALS IN MULTIPLE TISSUES AND 7126 05:15:37,000 --> 05:15:39,440 ORGANS GOING BACK 14 YEARS AND 7127 05:15:39,440 --> 05:15:42,080 THIS IS SHATTERING AND DEMANDS 7128 05:15:42,080 --> 05:15:43,400 WHEN TO THINK TO START TREATMENT 7129 05:15:43,400 --> 05:15:46,080 AND I HAVE THREE CHILDREN THAT 7130 05:15:46,080 --> 05:15:49,120 MAY OR MAY INTO THE CARRY THE 7131 05:15:49,120 --> 05:15:51,720 CARRIER AND I SHUTTER TO THINK 7132 05:15:51,720 --> 05:15:55,360 ABOUT THE YEAR-LONG PROCESS OF 7133 05:15:55,360 --> 05:15:56,040 NEURODEGENERATION TO THINK THEY 7134 05:15:56,040 --> 05:15:59,560 HAVE TO WAIT UNTIL THEY INTEREST 7135 05:15:59,560 --> 05:16:00,760 TROUBLE BEFORE GETTING 7136 05:16:00,760 --> 05:16:01,360 TREATMENT. 7137 05:16:01,360 --> 05:16:02,760 THIS WAIT SEEMS CRUEL AND UNJUST 7138 05:16:02,760 --> 05:16:05,880 AT THIS JUNCTURE. 7139 05:16:05,880 --> 05:16:09,800 I URGE THIS ESTEEMED GROUP TO 7140 05:16:09,800 --> 05:16:11,880 TREAT THOSE WITH BIOMARKERS OR 7141 05:16:11,880 --> 05:16:14,640 MUTATIONS SOONER AS A TOP 7142 05:16:14,640 --> 05:16:16,360 OBJECTIVE IN THE ALS STRATEGIC 7143 05:16:16,360 --> 05:16:16,560 PLAN. 7144 05:16:16,560 --> 05:16:18,280 THIS HAS TO BE FIRST AND 7145 05:16:18,280 --> 05:16:19,360 FOREMOST IN MY VIEW DRIVING 7146 05:16:19,360 --> 05:16:21,760 EVERYTHING ELSE WITHIN THE PLAN. 7147 05:16:21,760 --> 05:16:32,120 THANK YOU, DEBORAH. 7148 05:16:32,760 --> 05:16:34,800 >>THE MONTH OF MAY IS ALS 7149 05:16:34,800 --> 05:16:40,240 AWARENESS MONTH ALS AND FOX 7150 05:16:40,240 --> 05:16:41,640 DON'T HAVE ANY NEWS ABOUT THE 7151 05:16:41,640 --> 05:16:47,120 ALS AWARENESS MONTH. 7152 05:16:47,120 --> 05:16:49,240 THE ORGANIZATIONS NEED TO MAKE 7153 05:16:49,240 --> 05:16:53,680 AN EFFORT IN GETTING THE WORD 7154 05:16:53,680 --> 05:16:55,680 OUT AND THE WRONG PEOPLE ARE 7155 05:16:55,680 --> 05:16:58,840 BEING TARGETED AND PEARL WITH 7156 05:16:58,840 --> 05:17:00,920 ALS ALREADY KNOW ABOUT ALS. 7157 05:17:00,920 --> 05:17:02,080 IT'S THE NON-ALS COMMUNITY THAT 7158 05:17:02,080 --> 05:17:12,600 NEEDS THE MESSAGE TENT TO THEM. 7159 05:17:16,800 --> 05:17:21,640 >>THE DOCUMENTED SUB GROUPS 7160 05:17:21,640 --> 05:17:25,760 SUGGEST THE RESEARCH IS CREATING 7161 05:17:25,760 --> 05:17:29,520 AN INEFFICIENT INFRASTRUCTURE TO 7162 05:17:29,520 --> 05:17:31,960 GATHER PATIENT DATA SUCH AS BIO 7163 05:17:31,960 --> 05:17:37,000 FLUIDS AND CLINICAL OBSERVATIONS 7164 05:17:37,000 --> 05:17:42,360 INTO THE LOOKING AT THE COURSE 7165 05:17:42,360 --> 05:17:43,480 OF THE DISEASE. 7166 05:17:43,480 --> 05:17:48,760 I HOPE AND PLEAD WITH NINDS THAT 7167 05:17:48,760 --> 05:17:56,960 THIS IS IS LOOKED AT IN THE 7168 05:17:56,960 --> 05:18:02,040 POPULATION EVERY FIVE YEARS. 7169 05:18:02,040 --> 05:18:04,280 RIGHT NOW IT SEEMS THE STRATEGIC 7170 05:18:04,280 --> 05:18:07,400 PLAN AND THE NEXT GENERATION OF 7171 05:18:07,400 --> 05:18:10,760 PATIENTS AND THE GOVERNMENT 7172 05:18:10,760 --> 05:18:12,480 ALTER IN FAVOR OF A LINEAR 7173 05:18:12,480 --> 05:18:22,640 PROFESSOR. 7174 05:18:26,080 --> 05:18:29,760 AND CONSIDERING NEEDS TO BE 7175 05:18:29,760 --> 05:18:40,040 GIVEN TO THE ALS CLOCK. 7176 05:18:40,040 --> 05:18:43,840 >>THIS IS FROM BARBARA KITT. 7177 05:18:43,840 --> 05:18:47,800 I'M A RETIRED BUSINESS EXECUTIVE 7178 05:18:47,800 --> 05:18:49,120 AND ETHICS OFFICER ADVOCATING 7179 05:18:49,120 --> 05:18:51,560 FOR ADVANCES IN ALS RESEARCH AND 7180 05:18:51,560 --> 05:18:54,440 TREATMENTS WITH THE ALS PROBLEM 7181 05:18:54,440 --> 05:18:57,040 SOLVERS FOR THE PAST TWO YEARS. 7182 05:18:57,040 --> 05:18:58,840 AS IT RELATES TO THE STRATEGIC 7183 05:18:58,840 --> 05:19:00,680 PLAN FOR RESEARCH, WE COMMEND 7184 05:19:00,680 --> 05:19:05,280 THE NIH FOR UNDERTAKING THE 7185 05:19:05,280 --> 05:19:07,200 EFFORT WE BELIEVE THE STRATEGIC 7186 05:19:07,200 --> 05:19:12,520 PRIORITIES ARE ALL IMPORTANT AND 7187 05:19:12,520 --> 05:19:16,160 GENERALLY MAP TO THE FIVE 7188 05:19:16,160 --> 05:19:16,760 COGS/SOLUTIONS PRESENTED AND 7189 05:19:16,760 --> 05:19:20,560 DISCUSSED WITH NINDS STAFF FIRST 7190 05:19:20,560 --> 05:19:24,240 IN OCTOBER 2021 AND SUBSEQUENT 7191 05:19:24,240 --> 05:19:25,440 MEETINGS IN 2022. 7192 05:19:25,440 --> 05:19:28,040 THANK YOU FOR DOING THIS. 7193 05:19:28,040 --> 05:19:29,480 IN YOUR DRAFT STRATEGIC PLAN WE 7194 05:19:29,480 --> 05:19:34,240 SEE AN ADMIRABLE LIST OF 15 7195 05:19:34,240 --> 05:19:34,560 PRIORITIES. 7196 05:19:34,560 --> 05:19:40,000 THEY'RE LIKE 15 FEATURES. 7197 05:19:40,000 --> 05:19:44,160 THERE'S OVERLAPS BETWEEN WE 7198 05:19:44,160 --> 05:19:45,760 BELIEVE SOME WORK STREAMS ARE 7199 05:19:45,760 --> 05:19:47,720 TOO MANY TO BE CONSIDERED 7200 05:19:47,720 --> 05:19:48,000 PRIORITIES. 7201 05:19:48,000 --> 05:19:51,160 THESE NOW NEED TO BE WOVEN INTO 7202 05:19:51,160 --> 05:19:55,760 A COHESIVE STRATEGIC PLAN IN THE 7203 05:19:55,760 --> 05:19:59,880 SAME WAY A SKILLED ARCHITECT 7204 05:19:59,880 --> 05:20:02,920 DESIGNS A HOME AND WE NEED A 7205 05:20:02,920 --> 05:20:05,240 FAST TRACKED TIME LINE THAT 7206 05:20:05,240 --> 05:20:07,120 RECOGNIZE THE ALS CLOCK. 7207 05:20:07,120 --> 05:20:08,680 WE CAN'T AFFORD DISPARATE 7208 05:20:08,680 --> 05:20:10,080 PRIORITIES THAT ARE NOT 7209 05:20:10,080 --> 05:20:11,960 IMPLEMENTED EFFICIENTLY AND THAT 7210 05:20:11,960 --> 05:20:14,480 WILL TAKE THE EXPERTISE AND 7211 05:20:14,480 --> 05:20:15,600 LEADERSHIP OF NINDS. 7212 05:20:15,600 --> 05:20:19,160 WE CAN'T AFFORD TO BUILD IT AND 7213 05:20:19,160 --> 05:20:20,720 THEY WILL COME APPROACH TO 7214 05:20:20,720 --> 05:20:24,400 INFRASTRUCTURE COMPONENTS. 7215 05:20:24,400 --> 05:20:27,560 A STRATEGIC PLAN NEEDS TO 7216 05:20:27,560 --> 05:20:30,480 CONSIDER THE IMPRACTICALITIES AN 7217 05:20:30,480 --> 05:20:32,600 ARCHITECT DEALS WITH IN DRAWING 7218 05:20:32,600 --> 05:20:33,800 UP SCHEMATICS FOR THEIR ENTIRE 7219 05:20:33,800 --> 05:20:35,880 MOMENT. 7220 05:20:35,880 --> 05:20:38,880 -- HOME. 7221 05:20:38,880 --> 05:20:41,080 >>THE NEXT COMMENT IS 7222 05:20:41,080 --> 05:20:41,440 ANONYMOUS. 7223 05:20:41,440 --> 05:20:43,200 I THINK THE ALS STRATEGIC PLAN 7224 05:20:43,200 --> 05:20:45,520 IS STILL IN ITS INFANCY TAKING 7225 05:20:45,520 --> 05:20:47,720 SMALL STEPS BUT NOT ENOUGH SINCE 7226 05:20:47,720 --> 05:20:50,160 ALS KILLS FASTER THAN A BABY CAN 7227 05:20:50,160 --> 05:20:51,000 BECOME AN ADULT. 7228 05:20:51,000 --> 05:20:57,000 IT SHOULD BE ON A WIDER GLOBAL 7229 05:20:57,000 --> 05:20:57,200 SCALE. 7230 05:20:57,200 --> 05:20:58,040 CELEBRITIES, SINGERS, SPORTS 7231 05:20:58,040 --> 05:21:03,160 PERSONS CAN BE THE FACE OF ALS. 7232 05:21:03,160 --> 05:21:08,360 THE COMBINATION OF DIFFERENT 7233 05:21:08,360 --> 05:21:09,800 THERAPEUTICS SHOULD BE TRIED 7234 05:21:09,800 --> 05:21:12,840 LIKE AMEX 0035 AND THE EXAMPLE 7235 05:21:12,840 --> 05:21:13,800 BEING AIDS THERAPY. 7236 05:21:13,800 --> 05:21:21,680 THANK YOU FOR THAT. 7237 05:21:21,680 --> 05:21:24,200 >>I HAVE JUST RETIRED FROM 7238 05:21:24,200 --> 05:21:26,440 PFIZER AFTER 35 YEARS IN R&D AND 7239 05:21:26,440 --> 05:21:28,120 MOST RECENTLY AS CHIEF 7240 05:21:28,120 --> 05:21:29,760 DEVELOPMENT OFFICER RESPONSIBLE 7241 05:21:29,760 --> 05:21:30,600 FOR ALL CLINICAL DEVELOPMENT AND 7242 05:21:30,600 --> 05:21:33,760 REGULATORY AFFAIRS. 7243 05:21:33,760 --> 05:21:35,360 I NOW VOLUNTEER AS THE ALS 7244 05:21:35,360 --> 05:21:37,560 PROBLEM SOLVERS. 7245 05:21:37,560 --> 05:21:39,000 I'VE BEEN INVOLVED IN THE ALS 7246 05:21:39,000 --> 05:21:42,800 PROBLEM SOLVER DISCUSSIONS THIS 7247 05:21:42,800 --> 05:21:45,240 YEAR AND I'M HAPPY TO SEE MUCH 7248 05:21:45,240 --> 05:21:47,880 OF THE FIVE COGS WE RECOMMENDED 7249 05:21:47,880 --> 05:21:50,960 ARE CENTRAL TO THE DRAFT 7250 05:21:50,960 --> 05:21:52,080 STRATEGY AND THEY DOES AN 7251 05:21:52,080 --> 05:21:54,120 EXCELLENT JOB OF EXPLAINING THE 7252 05:21:54,120 --> 05:21:55,600 NATURAL HISTORY STUDY FOR THEIR 7253 05:21:55,600 --> 05:22:00,080 WORK AND SAW THE NEED FOR SUCH 7254 05:22:00,080 --> 05:22:03,000 LONGITUDINAL INTEROPERABLE DATA. 7255 05:22:03,000 --> 05:22:04,640 A NATURAL HISTORY STUDY WAS 7256 05:22:04,640 --> 05:22:05,240 SPECIFICALLY MENTIONED NINE 7257 05:22:05,240 --> 05:22:13,800 TIMES AND REFERENCES TO THE DATA 7258 05:22:13,800 --> 05:22:16,240 AND IT IS CLEAR THAT THEREFORE 7259 05:22:16,240 --> 05:22:19,360 THE NATURAL HISTORY STUDY IS 7260 05:22:19,360 --> 05:22:20,800 FOUNDATIONAL TO MULTIPLE 7261 05:22:20,800 --> 05:22:22,080 PRIORITIES IN THE DRAFT STRATEGY 7262 05:22:22,080 --> 05:22:23,240 DOCUMENT AND SHOULD BE 7263 05:22:23,240 --> 05:22:25,440 IMPLEMENTED IN A MANNER TO 7264 05:22:25,440 --> 05:22:28,240 PROVIDE BROAD ACCESS TO A 7265 05:22:28,240 --> 05:22:29,800 REPRESENTATIVE POPULATION OF 7266 05:22:29,800 --> 05:22:35,880 PEOPLE LIVING WITH ALS. 7267 05:22:35,880 --> 05:22:36,960 IT'S CRITICAL DESIGN CRITERIA 7268 05:22:36,960 --> 05:22:42,520 ARE FINALIZED AND THE STUDY 7269 05:22:42,520 --> 05:22:44,400 STARTED AS SOON AS POSSIBLE AND 7270 05:22:44,400 --> 05:22:45,960 SHOULD BE OPERATIONAL AND 7271 05:22:45,960 --> 05:22:51,400 QUALITY AND SPEED SHOULD DEFINE 7272 05:22:51,400 --> 05:22:56,600 THE TIME LINE NOT PROCESS AND 7273 05:22:56,600 --> 05:22:58,480 EVERY MINUTE MATTERS. 7274 05:22:58,480 --> 05:23:02,360 >>THE NEXT ITEM IS AN ANONYMOUS 7275 05:23:02,360 --> 05:23:07,280 COMMENT ORIGINALLY IN FRENCH AND 7276 05:23:07,280 --> 05:23:07,520 TRANSLATE. 7277 05:23:07,520 --> 05:23:09,040 WE'RE A DOUBLE GLOBAL C9 7278 05:23:09,040 --> 05:23:11,160 COMMUNITY AND YOU KNOW OUR 7279 05:23:11,160 --> 05:23:12,040 STARTS AND NOTHING IS DONE AND 7280 05:23:12,040 --> 05:23:14,160 WILLING TO BE VOLUNTEERS. 7281 05:23:14,160 --> 05:23:16,040 WHY DON'T WE TEST EXISTING DRUGS 7282 05:23:16,040 --> 05:23:19,320 THAT DON'T WORK ONCE THE DISEASE 7283 05:23:19,320 --> 05:23:21,560 BUT TEST THEM BEFORE THE DISEASE 7284 05:23:21,560 --> 05:23:27,080 WHETHER FTD OR ALS DEVELOPS 7285 05:23:27,080 --> 05:23:32,160 THANK YOU FOR THE QUESTION. 7286 05:23:32,160 --> 05:23:42,640 >>THE COMMENT IS FROM AN A 7287 05:23:49,840 --> 05:23:51,480 WOMAN WHO'S SPOUSE PASSED AND 7288 05:23:51,480 --> 05:23:55,360 I'VE BEEN INVOLVED IN ADVOCACY 7289 05:23:55,360 --> 05:23:56,680 FOR 17 YEARS. 7290 05:23:56,680 --> 05:23:58,000 MOSTLY AS PART OF THE ALS 7291 05:23:58,000 --> 05:24:03,880 PROBLEM SOLVERS. 7292 05:24:03,880 --> 05:24:10,320 WE SALUTE NINDS FOR THOSE 7293 05:24:10,320 --> 05:24:12,520 SUPPORTING PATIENTS AND 7294 05:24:12,520 --> 05:24:14,120 CAREGIVERS ON THE STRATEGIC 7295 05:24:14,120 --> 05:24:17,560 PLANNING AND WE EXPECT THE 7296 05:24:17,560 --> 05:24:21,040 REPRESENTATION TO CONTINUE AS A 7297 05:24:21,040 --> 05:24:23,840 PLAN IS IMPLEMENTED. 7298 05:24:23,840 --> 05:24:26,600 I NOTICED PAGE 29 SAYS, AND I 7299 05:24:26,600 --> 05:24:29,560 QUOTE, FORMATION OF A COMMITTEE 7300 05:24:29,560 --> 05:24:33,800 THAT INCLUDES REPRESENTATION 7301 05:24:33,800 --> 05:24:37,680 FROM ACADEMIA AND NONPROFIT AND 7302 05:24:37,680 --> 05:24:42,880 GOVERNMENT FUNDING AGENCIES WITH 7303 05:24:42,880 --> 05:24:47,520 A FOCUS ON THE GOALS AND 7304 05:24:47,520 --> 05:24:52,760 RESOURCES FROM DIVERSE 7305 05:24:52,760 --> 05:24:54,560 CATEGORIES ALONG WITH PROTOCOLS 7306 05:24:54,560 --> 05:24:57,800 AND METHODOLOGIES FOR THE 7307 05:24:57,800 --> 05:24:58,640 GENERATIONAL USE. 7308 05:24:58,640 --> 05:24:59,880 END QUOTE. 7309 05:24:59,880 --> 05:25:05,760 I WOULD IMPLORE THAT PEOPLE 7310 05:25:05,760 --> 05:25:12,800 PEOPLE LIVING WITH ALS BE 7311 05:25:12,800 --> 05:25:13,800 REPRESENTATI 7312 05:25:13,800 --> 05:25:14,480 REPRESENTED IN THE COMMUNITY AND 7313 05:25:14,480 --> 05:25:17,800 THEY'RE THE EXPERTS AS THEY'RE 7314 05:25:17,800 --> 05:25:18,560 LIVING ON THE ALS CAR. 7315 05:25:18,560 --> 05:25:21,520 THANK YOU FOR THIS CHANCE TO 7316 05:25:21,520 --> 05:25:21,720 COMMENT. 7317 05:25:21,720 --> 05:25:28,440 WE THINK THE ALS PROBLEM SOLVERS 7318 05:25:28,440 --> 05:25:33,240 AND PEOPLE LIVING WITH ALS HAVE 7319 05:25:33,240 --> 05:25:41,200 SHOWN HOW HARD THEY'LL WORK TO 7320 05:25:41,200 --> 05:25:47,880 DELIVER ON THE MISSION. 7321 05:25:47,880 --> 05:25:49,160 AND PROVIDE AN A COMMUNITY OF 7322 05:25:49,160 --> 05:25:50,320 OUTREACH AND EDUCATION. 7323 05:25:50,320 --> 05:25:53,920 PUT US TO WORK WITH YOU. 7324 05:25:53,920 --> 05:25:57,760 WE CAN AND WILL HELP. 7325 05:25:57,760 --> 05:26:08,040 THANK YOU, JIM. 7326 05:26:10,920 --> 05:26:17,680 >>THIS IS FROM SOMEONE WHO'S 7327 05:26:17,680 --> 05:26:21,160 PARTNER PASSED FROM ALS. 7328 05:26:21,160 --> 05:26:23,600 76 DAYS AFTER DIAGNOSIS. 7329 05:26:23,600 --> 05:26:27,080 THANK YOU FOR PUTTING THE 7330 05:26:27,080 --> 05:26:28,120 STRATEGIC PLAN TOGETHER AND 7331 05:26:28,120 --> 05:26:29,920 THERE WAS NO TIME TO PLAN WITH 7332 05:26:29,920 --> 05:26:31,520 ME AS HER ONLY CAREGIVER WE 7333 05:26:31,520 --> 05:26:32,800 MOVED FORWARD WITH NO EQUIPMENT, 7334 05:26:32,800 --> 05:26:34,920 NO SKILLED NURSING CARE AND NO 7335 05:26:34,920 --> 05:26:35,880 REAL KNOWLEDGE AT HOW TO MANAGE 7336 05:26:35,880 --> 05:26:41,400 THE MEDICAL SYSTEM. 7337 05:26:41,400 --> 05:26:42,640 THIS STRATEGIC PLAN FOR 7338 05:26:42,640 --> 05:26:45,160 IMPLEMENTATION OF THE PLAN WITH 7339 05:26:45,160 --> 05:26:47,880 IMPLEMENTATION TO BEGIN IN LATE 7340 05:26:47,880 --> 05:26:50,720 2024 IS NOT AGGRESSIVE ENOUGH. 7341 05:26:50,720 --> 05:26:52,680 IT DOES NOT REFLECT THE ALS 7342 05:26:52,680 --> 05:26:52,880 CLOCK. 7343 05:26:52,880 --> 05:26:54,120 FOR US THE CLOCK WAS LESS THAN 7344 05:26:54,120 --> 05:26:54,880 THREE MONTHS. 7345 05:26:54,880 --> 05:26:57,120 IT WILL BE OVER TWO YEARS FROM 7346 05:26:57,120 --> 05:26:58,320 THE TIME THE STRATEGIC PLANNING 7347 05:26:58,320 --> 05:27:01,760 EFFORT WAS ANNOUNCED TO 7348 05:27:01,760 --> 05:27:05,000 BEBEGINNING -- BEGINNING OF THE 7349 05:27:05,000 --> 05:27:06,360 IMPLEMENTATION AND DURING THAT 7350 05:27:06,360 --> 05:27:08,080 TIME OVER 12,000 PEOPLE LIVING 7351 05:27:08,080 --> 05:27:09,280 WITH ALS WILL DIE AND MANY WILL 7352 05:27:09,280 --> 05:27:12,600 LOSE THEIR ABILITY TO WALK, 7353 05:27:12,600 --> 05:27:14,880 TALK, FEED AND PERFORM BASIC 7354 05:27:14,880 --> 05:27:16,960 DAILY LIFE FUNCTIONS. 7355 05:27:16,960 --> 05:27:21,120 NIH SHOULD AGGRESSIVELY ACC 7356 05:27:21,120 --> 05:27:24,440 ACCELERATE THE TIME LINE, WHAT 7357 05:27:24,440 --> 05:27:25,840 CAN I DO TO SUPPORT NIH IN 7358 05:27:25,840 --> 05:27:28,760 ACCELERATING THIS PLAN? 7359 05:27:28,760 --> 05:27:31,920 WHAT CAN WE THE ALS COMMUNITY DO 7360 05:27:31,920 --> 05:27:34,920 TO SUPPORT NIH IN ACCELERATING 7361 05:27:34,920 --> 05:27:35,120 THIS. 7362 05:27:35,120 --> 05:27:36,440 WHAT CAN NIH DO TO ACCELERATE 7363 05:27:36,440 --> 05:27:41,880 THE IMPLEMENTATION OF THE K 7364 05:27:41,880 --> 05:27:42,200 COHESIVE PLAN. 7365 05:27:42,200 --> 05:27:52,400 THANK YOU. 7366 05:27:52,960 --> 05:27:57,080 >>AND GIVEN THE STUDY OF 7367 05:27:57,080 --> 05:28:01,720 DECLINE AND JAPAN AND INDONESIA 7368 05:28:01,720 --> 05:28:04,240 AND GUAM AND POST-WORLD WAR II 7369 05:28:04,240 --> 05:28:07,200 AND THE DISTINCT GENETIC GROUPS 7370 05:28:07,200 --> 05:28:09,640 NONE OF THOSE WITH A GENETIC 7371 05:28:09,640 --> 05:28:11,320 PHENOTYPE AND IT'S THOUGHT 7372 05:28:11,320 --> 05:28:15,120 EXOGENOUS FACTORS CAN PLAY AN 7373 05:28:15,120 --> 05:28:25,640 EXCLUSIVE ET YO LODGE -- 7374 05:28:31,880 --> 05:28:34,160 ETIOLOGIC ROLE AND THERE'S A HOT 7375 05:28:34,160 --> 05:28:37,760 SPOT IN THE FRENCH ALPS WHERE 7376 05:28:37,760 --> 05:28:41,880 FUNGI CONTAINING CHEMICALS 7377 05:28:41,880 --> 05:28:43,720 COMPARED WITH THOSE IN THE 7378 05:28:43,720 --> 05:28:45,760 WESTERN PACIFIC ALS. 7379 05:28:45,760 --> 05:28:48,480 THE DISCOVERY OF THIS CAN LEAD 7380 05:28:48,480 --> 05:28:51,040 TO PRIMARY ALS PREVENTION 7381 05:28:51,040 --> 05:28:53,800 UNDERSTANDING CAUSE-ASSOCIATED 7382 05:28:53,800 --> 05:28:55,840 MECHANISMS CAN LEAD TO BIOMARKER 7383 05:28:55,840 --> 05:28:56,400 DISCOVERY AND THERAPEUTIC 7384 05:28:56,400 --> 05:28:56,720 DEVELOPMENT. 7385 05:28:56,720 --> 05:29:04,440 THANK YOU, PETER. 7386 05:29:04,440 --> 05:29:07,680 >>THE NEXT COMMENT. 7387 05:29:07,680 --> 05:29:09,080 I GOT INVOLVED IN ADVOCACY WHEN 7388 05:29:09,080 --> 05:29:11,160 MY FRIEND WAS DIAGNOSED WITH THE 7389 05:29:11,160 --> 05:29:16,080 DISEASE IN EARLY 2021. 7390 05:29:16,080 --> 05:29:17,760 SADLY, SHE PASSED NINE MONTHS 7391 05:29:17,760 --> 05:29:19,480 LATER AND THE ALS CLOCK MOVES 7392 05:29:19,480 --> 05:29:20,560 FAST. 7393 05:29:20,560 --> 05:29:22,360 I'M HAPPY NIH IS PUTTING 7394 05:29:22,360 --> 05:29:23,600 TOGETHER A STRATEGY PLAN TO 7395 05:29:23,600 --> 05:29:25,360 ADVANCE RESEARCH, THERAPIES AND 7396 05:29:25,360 --> 05:29:28,960 CURE FOR ALS. 7397 05:29:28,960 --> 05:29:30,200 I WONDER HOW QUICKLY ELEMENTS OF 7398 05:29:30,200 --> 05:29:32,040 THE PLAN WILL TAKE AND THE 7399 05:29:32,040 --> 05:29:33,760 OVERLAP OF SOME ACTIVITIES 7400 05:29:33,760 --> 05:29:34,800 ESPECIALLY RELATED TO NATURAL 7401 05:29:34,800 --> 05:29:37,760 HISTORY AND THE NEED FOR 7402 05:29:37,760 --> 05:29:45,760 LONGITUDINAL DATA. 7403 05:29:45,760 --> 05:29:48,320 A 7404 05:29:48,320 --> 05:29:50,200 ARE THERE FUNDS AT NIH TO BEGIN 7405 05:29:50,200 --> 05:29:52,720 EFFORTS IMMEDIATELY OR WILL YOU 7406 05:29:52,720 --> 05:29:54,640 HAVE TO WAIT UNTIL 2024 WHEN 7407 05:29:54,640 --> 05:29:57,200 CONGRESS CAN APPROPRIATE FUNDS? 7408 05:29:57,200 --> 05:29:58,560 MANY MUCH THE FRIENDS WITH ALS 7409 05:29:58,560 --> 05:29:59,920 DON'T HAVE TIME TO WAIT UNTIL 7410 05:29:59,920 --> 05:30:06,560 2024 TO SEE ACTION. 7411 05:30:06,560 --> 05:30:10,240 WE NEED TO WORK WITH SPEED AND 7412 05:30:10,240 --> 05:30:10,480 URGENCY. 7413 05:30:10,480 --> 05:30:20,800 LIVES DEPEND ON IT. 7414 05:30:23,200 --> 05:30:25,440 >>THERE'S BEEN TIME AND EFFORT 7415 05:30:25,440 --> 05:30:26,960 BY THE FIVE WORKING GROUPS TO 7416 05:30:26,960 --> 05:30:28,960 THANK YOU TO EACH MEMBER. 7417 05:30:28,960 --> 05:30:31,600 ONE COMMON THEME THROUGHOUT THE 7418 05:30:31,600 --> 05:30:33,040 DRAFT IS DATA COLLECTION. 7419 05:30:33,040 --> 05:30:35,680 DESCRIBED IN WORKING GROUP THREE 7420 05:30:35,680 --> 05:30:38,720 AS A NATURAL HISTORY STUDY. 7421 05:30:38,720 --> 05:30:41,120 OTHERS PRESCRIBE DATA COLLECTION 7422 05:30:41,120 --> 05:30:44,720 OF BIO SAMPLES AND THIS 7423 05:30:44,720 --> 05:30:45,440 LONGITUDINAL NATURAL HISTORY 7424 05:30:45,440 --> 05:30:47,080 STUDY SHOULD BE THE TOP PRIORITY 7425 05:30:47,080 --> 05:30:48,080 OF THE STRATEGIC PLAN BECAUSE IT 7426 05:30:48,080 --> 05:30:49,480 REPRESENT THE LARGEST POPULATION 7427 05:30:49,480 --> 05:30:51,320 OF PEOPLE LIVING WITH ALS. 7428 05:30:51,320 --> 05:30:53,760 AND A LARGE NUMBER OF WORKING 7429 05:30:53,760 --> 05:30:55,080 GROUP PRIORITIES NATURALLY FIT 7430 05:30:55,080 --> 05:30:58,760 AND PROCEED FROM IT. 7431 05:30:58,760 --> 05:31:01,360 ONE AREA I THINK THE STRATEGIC 7432 05:31:01,360 --> 05:31:02,760 PLAN RECOGNIZES BUT FAILS TO 7433 05:31:02,760 --> 05:31:04,200 PROVIDE AN ADEQUATE SOLUTION FOR 7434 05:31:04,200 --> 05:31:06,520 IS THAT OF DIVERSITY, EQUITY AND 7435 05:31:06,520 --> 05:31:06,800 INCLUSION. 7436 05:31:06,800 --> 05:31:09,320 THE AREAS PEOPLE ARE BEING LEFT 7437 05:31:09,320 --> 05:31:12,640 OUT ARE RURAL AND INTERCITY AND 7438 05:31:12,640 --> 05:31:13,640 SOCIO ECONOMICALLY ADVANTAGED 7439 05:31:13,640 --> 05:31:13,840 GROUPS. 7440 05:31:13,840 --> 05:31:17,560 ACROSS THE U.S., WE HAVE A BROAD 7441 05:31:17,560 --> 05:31:19,680 NETWORK OF MULTI-DISCIPLINARY 7442 05:31:19,680 --> 05:31:21,040 CLINICS AND THOSE LEFT BEHIND 7443 05:31:21,040 --> 05:31:21,760 AREN'T ABLE TO ACCESS THE 7444 05:31:21,760 --> 05:31:23,920 EXISTING RESOURCES. 7445 05:31:23,920 --> 05:31:26,520 PROPOSING TO USE THE EXISTING 7446 05:31:26,520 --> 05:31:27,920 NETWORK OR DESIGNATE SOME OF 7447 05:31:27,920 --> 05:31:30,280 THEM AS RESEARCH CENTERS OF 7448 05:31:30,280 --> 05:31:32,080 EXCELLENCE DOESN'T SOLVE THE 7449 05:31:32,080 --> 05:31:32,360 PROBLEM. 7450 05:31:32,360 --> 05:31:33,840 I WOULD SUGGEST REACHING OUT TO 7451 05:31:33,840 --> 05:31:35,920 INDIVIDUAL SINGLE PRACTICE 7452 05:31:35,920 --> 05:31:37,560 NEUROLOGISTS WHO TREAT ALS 7453 05:31:37,560 --> 05:31:41,320 PATIENTS AS WELL AS INCORPORATE 7454 05:31:41,320 --> 05:31:42,320 BEING ENVIRONMENT CLINICAL 7455 05:31:42,320 --> 05:31:44,360 RESOURCES THAT ANYONE DIAGNOSED 7456 05:31:44,360 --> 05:31:45,000 WITH OR THINK MAY HAVE ALS CAN 7457 05:31:45,000 --> 05:31:51,320 ACCESS. 7458 05:31:51,320 --> 05:31:52,480 ADDING NEW CLINICS WILL HELP. 7459 05:31:52,480 --> 05:31:55,000 THE GOAL IS TO BROADEN THE 7460 05:31:55,000 --> 05:31:56,520 PATIENT POPULATION SO ALL CAN 7461 05:31:56,520 --> 05:31:58,960 RECEIVE CARE AND PARTICIPATE IN 7462 05:31:58,960 --> 05:32:01,080 THE LANDMARK NATURAL HISTORY 7463 05:32:01,080 --> 05:32:02,680 STUDY AND CLINICAL TRIALS AND 7464 05:32:02,680 --> 05:32:04,120 MORE SPECIALIZED STUDIES 7465 05:32:04,120 --> 05:32:05,920 INVOLVING DATA COLLECTION OF BIO 7466 05:32:05,920 --> 05:32:08,160 SAMPLES AND OTHER USEFUL DATA TO 7467 05:32:08,160 --> 05:32:09,920 LEARN AS MUCH AS WE CAN ABOUT 7468 05:32:09,920 --> 05:32:17,760 ALS AND ITS CAUSES AND A CURE. 7469 05:32:17,760 --> 05:32:21,520 THANK YOU VERY MUCH, GLEN. 7470 05:32:21,520 --> 05:32:23,920 >>AND THIS ONE SAYS I WAS 7471 05:32:23,920 --> 05:32:29,880 DIAGNOSED IN AUGUST OF 2018. 7472 05:32:29,880 --> 05:32:33,320 I'M AN ADVOCATE FOR A MORE 7473 05:32:33,320 --> 05:32:36,760 INCLUSIVE CLINICAL TRIALS IN 7474 05:32:36,760 --> 05:32:37,760 ALS. 7475 05:32:37,760 --> 05:32:39,840 THE CENTERS OF EXCELLENCE 7476 05:32:39,840 --> 05:32:44,960 PROGRAM AND ALS RESEARCH AS 7477 05:32:44,960 --> 05:32:55,000 DESCRIBED AND AND THERE'S WELL 7478 05:32:55,000 --> 05:32:59,720 ESTABLISHED MULTI-DISCIPLINARY 7479 05:32:59,720 --> 05:33:04,520 ALS FUND IING AND IT'S COUNTER 7480 05:33:04,520 --> 05:33:12,880 PRODUCTIVE WHEN IT COMES 7481 05:33:12,880 --> 05:33:13,880 DIVERSITY EQUITY AND INCLUSION 7482 05:33:13,880 --> 05:33:16,520 AND MAY MAYBE IT HARDER, NOT 7483 05:33:16,520 --> 05:33:20,080 EASIER, TO STUDY THE NATURAL 7484 05:33:20,080 --> 05:33:21,280 HISTORY OF ALS AS IS ONLY LOOKED 7485 05:33:21,280 --> 05:33:30,640 AT A SUBSET OF THE POPULATION 7486 05:33:30,640 --> 05:33:33,760 AND THE CENTERS OF EXCELLENCE 7487 05:33:33,760 --> 05:33:35,920 COULD BE GEOGRAPHICALLY OR 7488 05:33:35,920 --> 05:33:39,440 SOCIALLY ECONOMICALLY 7489 05:33:39,440 --> 05:33:40,440 EXCLUSIONARY. 7490 05:33:40,440 --> 05:33:47,560 THE DEI PROBLEM IS DESCRIBED BUT 7491 05:33:47,560 --> 05:33:48,160 NO TANGIBLE SOLUTIONS WERE 7492 05:33:48,160 --> 05:33:53,800 OFFERED. 7493 05:33:53,800 --> 05:33:57,480 PERHAPS A PROGRAM COULD INCLUDE 7494 05:33:57,480 --> 05:34:08,000 HBCUs AND MEDICAL SCHOOLS AND 7495 05:34:10,720 --> 05:34:12,200 HOW DO WE EXPAND THIS IN THE 7496 05:34:12,200 --> 05:34:13,760 U.S.? 7497 05:34:13,760 --> 05:34:22,600 WE NEED TO DO MORE THAN THE DEI 7498 05:34:22,600 --> 05:34:22,840 PROBLEM. 7499 05:34:22,840 --> 05:34:31,840 WE NEED AND THOSE NOT TREATED IN 7500 05:34:31,840 --> 05:34:33,840 ESTABLISHED CLINICS OR BY 7501 05:34:33,840 --> 05:34:36,520 PROMINENT CLINICIANS NEED TO BE 7502 05:34:36,520 --> 05:34:46,920 REACHED AND INCLUDED. 7503 05:34:47,760 --> 05:34:50,320 AND WE MUST HAVE DECENTRALIZED 7504 05:34:50,320 --> 05:34:51,200 CLINICAL TRIALS THAT CAN BE USED 7505 05:34:51,200 --> 05:34:53,760 TO OVERCOME THE BARRIERS. 7506 05:34:53,760 --> 05:35:03,960 THANK YOU. 7507 05:35:04,640 --> 05:35:11,560 >>THE COMMENT IS FROM 7508 05:35:11,560 --> 05:35:15,680 JEAN-PIERRE. 7509 05:35:15,680 --> 05:35:17,760 AS SOMEONE WHO HAD TWO PEOPLE IN 7510 05:35:17,760 --> 05:35:20,960 ALS IN MY FAMILY I THINK 7511 05:35:20,960 --> 05:35:23,680 RESEARCH SHOULD ADOPT A TOOL 7512 05:35:23,680 --> 05:35:27,480 FAMOUS TO THE HALLMARKS OF 7513 05:35:27,480 --> 05:35:27,720 CANCER. 7514 05:35:27,720 --> 05:35:30,320 A TENTATIVE CHECKLIST WOULD BE 7515 05:35:30,320 --> 05:35:35,400 TO ASK, 1, DOES IT OFFER A 7516 05:35:35,400 --> 05:35:36,240 COMPELLING EXPLANATION MUSCLE 7517 05:35:36,240 --> 05:35:38,360 WASTING BASED ON METABOLISM AND 7518 05:35:38,360 --> 05:35:41,880 NOT PROLONGED INACTIVITY OF 7519 05:35:41,880 --> 05:35:42,600 UPPER NEURONS. 7520 05:35:42,600 --> 05:35:44,480 2, DOES IT EXPLAIN WHY SYMPTOMS 7521 05:35:44,480 --> 05:35:47,520 OFTEN START IN A SPECIFIC AREA? 7522 05:35:47,520 --> 05:35:52,840 3, DOES IT EXTEND WHY SYMPTOMS 7523 05:35:52,840 --> 05:35:57,040 EXTEND PROGRESSIVELY? 7524 05:35:57,040 --> 05:35:59,200 4, DOES IT EXPLAIN WHY IT 7525 05:35:59,200 --> 05:36:01,760 STRIKES THE ELDERLY. 7526 05:36:01,760 --> 05:36:03,960 5, DOES IT EXPLAIN WHY SOME 7527 05:36:03,960 --> 05:36:06,600 SURVIVE 10 TO 20 YEARS AND SOME 7528 05:36:06,600 --> 05:36:07,680 SIX MONTHS? 7529 05:36:07,680 --> 05:36:11,240 6, DOES IT EXPLAIN WHY ONLY 7530 05:36:11,240 --> 05:36:21,760 SKELETAL MUSCLES ARE IMPACTED? 7531 05:36:21,760 --> 05:36:25,120 7, DOES IT EXPLAIN WHY ONLY 7532 05:36:25,120 --> 05:36:27,640 HIGHER PRIMATES SEEM TO HAVE 7533 05:36:27,640 --> 05:36:28,840 ALS-LIKE DISEASES? 7534 05:36:28,840 --> 05:36:30,840 ANOTHER THOUGHT IS THAT RESEARCH 7535 05:36:30,840 --> 05:36:33,240 ABOUT MANAGING THE DISEASE IS 7536 05:36:33,240 --> 05:36:35,320 LACKING, FOR EXAMPLE, I THINK 7537 05:36:35,320 --> 05:36:37,760 ALS SCIENTISTS HAS A LOT TO 7538 05:36:37,760 --> 05:36:39,320 LEARN FROM STROKE AND FINAL 7539 05:36:39,320 --> 05:36:40,960 INJURY SCIENTISTS AND FINALLY IT 7540 05:36:40,960 --> 05:36:43,240 WOULD BE DESIRABLE TO FUND 7541 05:36:43,240 --> 05:36:45,000 SCIENTISTS WITH BACKGROUNDS IN 7542 05:36:45,000 --> 05:36:49,040 HUMAN PHYSIOLOGY AND ALS 7543 05:36:49,040 --> 05:36:56,400 METABOLISM AND THE CORTICOBULBO 7544 05:36:56,400 --> 05:36:59,680 TRACK RATHER THAN SPECIALISTS IN 7545 05:36:59,680 --> 05:37:03,240 MOLECULAR BIOLOGY WHICH SEEM 7546 05:37:03,240 --> 05:37:04,360 UNABLE TO HELP PROGRESS BEYOND 7547 05:37:04,360 --> 05:37:04,800 LOW-HANGING FRUITS. 7548 05:37:04,800 --> 05:37:13,400 THANK YOU. 7549 05:37:13,400 --> 05:37:14,920 >>MUCH DISEASE FOCUSSED 7550 05:37:14,920 --> 05:37:18,440 RESEARCH AIMS TO JUNG TO THE 7551 05:37:18,440 --> 05:37:21,160 CURES WITHOUT RESTING ON 7552 05:37:21,160 --> 05:37:22,320 NEUROSCIENCE UNDERSTANDING OF 7553 05:37:22,320 --> 05:37:23,640 MOTOR NEURO BIOLOGY AND 7554 05:37:23,640 --> 05:37:23,920 FUNCTION. 7555 05:37:23,920 --> 05:37:25,480 I BELIEVE WE MUST MAKE IT CLEAR 7556 05:37:25,480 --> 05:37:27,720 THE LACK OF TREATMENT FOR ALS 7557 05:37:27,720 --> 05:37:31,840 ARE INHERENTLY LIMITED BY A POOR 7558 05:37:31,840 --> 05:37:33,840 UNDERSTANDING OF MOTOR NEURON 7559 05:37:33,840 --> 05:37:37,760 GENE REGULATION AND FEATURES OF 7560 05:37:37,760 --> 05:37:41,160 NEUROBIOLOGY AND ASPECTS OF RNA 7561 05:37:41,160 --> 05:37:41,480 REGULATION. 7562 05:37:41,480 --> 05:37:44,480 A NEW VISION FOR AN ALS 7563 05:37:44,480 --> 05:37:47,600 TREATMENT PIPELINE MUST CLEARLY 7564 05:37:47,600 --> 05:37:48,200 EMPHASIS BASIC NEUROSCIENCE 7565 05:37:48,200 --> 05:37:51,080 GOALS AND UNANSWERED QUESTIONS. 7566 05:37:51,080 --> 05:37:53,080 I DO NOT SEE SUCH A VISION IN 7567 05:37:53,080 --> 05:37:53,960 THE CURRENT FORM OF THE 7568 05:37:53,960 --> 05:37:54,440 STRATEGIC PLAN. 7569 05:37:54,440 --> 05:37:57,600 WHERE IS THE DISCUSSION OF THE 7570 05:37:57,600 --> 05:37:58,920 BASIC NEUROSCIENCE OBJECTIVES 7571 05:37:58,920 --> 05:38:00,120 THAT WOULD ADD TO THE NEEDS OF 7572 05:38:00,120 --> 05:38:05,760 THE BROADER ALS COMMUNITY? 7573 05:38:05,760 --> 05:38:13,280 THANK YOU, NEIL. 7574 05:38:13,280 --> 05:38:16,640 >>MOM DIED FROM ALS 25 YEARS 7575 05:38:16,640 --> 05:38:16,800 AGO. 7576 05:38:16,800 --> 05:38:17,520 I'VE BEEN INVOLVED IN ALS 7577 05:38:17,520 --> 05:38:18,400 ADVOCACY WORK SINCE. 7578 05:38:18,400 --> 05:38:21,080 I'M A MEMBER OF THE ALS PROBLEM 7579 05:38:21,080 --> 05:38:21,320 SOLVERS. 7580 05:38:21,320 --> 05:38:24,040 AS A GROUP WE FIRST MET WITH 7581 05:38:24,040 --> 05:38:26,880 NINDS LAST OCTOBER. 7582 05:38:26,880 --> 05:38:28,520 WE'RE VERY HAPPY NINDS HAS 7583 05:38:28,520 --> 05:38:29,760 UNDERTAKEN THE STRATEGIC PLAN 7584 05:38:29,760 --> 05:38:31,480 FOR ALS. 7585 05:38:31,480 --> 05:38:33,200 THERE'S ONLY ONE REFERENCE TO 7586 05:38:33,200 --> 05:38:35,960 EXPANDED ACCESS IN THE DRAFT. 7587 05:38:35,960 --> 05:38:39,000 EXPANDED ACCESS FOR ALS 7588 05:38:39,000 --> 05:38:42,200 RESOURCES SHOULD NOT LIVE IN A 7589 05:38:42,200 --> 05:38:42,680 SILO. 7590 05:38:42,680 --> 05:38:46,160 IF WE PUT PEOPLE LIVING WITH ALS 7591 05:38:46,160 --> 05:38:49,080 AT THE CENTER OF THE STRATEGIC 7592 05:38:49,080 --> 05:38:53,960 PLAN THEY SHOULD HAVE A ROLE IN 7593 05:38:53,960 --> 05:38:56,160 MOVING THE SCIENCE FORWARD AND 7594 05:38:56,160 --> 05:38:57,760 FOR THOSE WHO DO NOT QUALIFY FOR 7595 05:38:57,760 --> 05:39:01,960 TRIALS, THE EXPANDED ACCESS PATH 7596 05:39:01,960 --> 05:39:04,560 SHOULD BE AVAILABLE AND USED TO 7597 05:39:04,560 --> 05:39:07,160 ITS BEST SCIENTIFIC PURPOSE. 7598 05:39:07,160 --> 05:39:08,800 I HOPE THIS MAY BE HELPFUL TO 7599 05:39:08,800 --> 05:39:11,240 THE TRANSLATIONAL GROUP AND 7600 05:39:11,240 --> 05:39:12,480 CLINICAL TRIAL TRIALS GROUP AND 7601 05:39:12,480 --> 05:39:13,440 THE GROUP ADDRESSING QUALITY OF 7602 05:39:13,440 --> 05:39:15,560 LIFE ISSUES. 7603 05:39:15,560 --> 05:39:15,960 THANK YOU, MARY. 7604 05:39:15,960 --> 05:39:17,760 >>THE NEXT COMMENT IS FROM 7605 05:39:17,760 --> 05:39:19,600 ASHLEY LEE. 7606 05:39:19,600 --> 05:39:21,400 THE CURRENT EFFORTS AND WORK 7607 05:39:21,400 --> 05:39:23,960 PERTAIN TO ALS AND THE RESULTING 7608 05:39:23,960 --> 05:39:24,880 COLLABORATION IS INCREDIBLE. 7609 05:39:24,880 --> 05:39:27,000 I KNOW WE STILL HAVE A LONG WAY 7610 05:39:27,000 --> 05:39:28,880 TO GO BUT COMPARED TO THE 7611 05:39:28,880 --> 05:39:30,880 EXTREMELY MINIMAL AMOUNTS OF 7612 05:39:30,880 --> 05:39:32,560 RESEARCH AND ADVOCACY EFFORTS I 7613 05:39:32,560 --> 05:39:34,440 EXPERIENCED IN , WHEN WE BEGAN 7614 05:39:34,440 --> 05:39:36,720 THE JOURNEY, THIS IS TRULY 7615 05:39:36,720 --> 05:39:37,680 INSPIRING AND ENCOURAGING. 7616 05:39:37,680 --> 05:39:40,680 THANK YOU. 7617 05:39:40,680 --> 05:39:45,720 >>THINK THIS IS THE LAST 7618 05:39:45,720 --> 05:39:47,400 PRE-SUBMITTED COMMENT AND IT 7619 05:39:47,400 --> 05:39:52,600 COMES FROM I HOPE I'M 7620 05:39:52,600 --> 05:40:03,200 PRONOUNCING THIS RIGHT, NEIL 7621 05:40:04,880 --> 05:40:05,040 RESNICK. 7622 05:40:05,040 --> 05:40:08,560 I LIVED FOR 10 YEARS WITH MY 7623 05:40:08,560 --> 05:40:14,640 HUSBAND'S ALS PARALYZED WITH A 7624 05:40:14,640 --> 05:40:20,400 RESPIRATOR AND WE RECEIVED VERY 7625 05:40:20,400 --> 05:40:22,120 LITTLE FINANCIAL SUPPORT AND HAD 7626 05:40:22,120 --> 05:40:28,600 TO FUND RAISE TO AFFORD TO PAY 7627 05:40:28,600 --> 05:40:39,120 CAREGIVERS AND LOANS FOR FOOD. 7628 05:40:39,840 --> 05:40:49,760 FOR THOSE WHO SURVIVE WHAT IS 7629 05:40:49,760 --> 05:40:53,640 IMPOSED BY RESEARCH NEEDS TO GO 7630 05:40:53,640 --> 05:40:54,800 BACK TO ALS RESEARCH. 7631 05:40:54,800 --> 05:41:00,720 IF WE WANT TO SURVIVE LONG TERM 7632 05:41:00,720 --> 05:41:03,160 UNTIL RESEARCH DISCOVERS A CURE 7633 05:41:03,160 --> 05:41:07,200 WE NEED MAJOR FUNDING FOR THE 7634 05:41:07,200 --> 05:41:09,760 CARE, SUPPORT OF THE FAMILY 7635 05:41:09,760 --> 05:41:20,120 CAREGIVERS THEMSELVES. 7636 05:41:21,720 --> 05:41:23,920 >>THERE'S A COUPLE COMMENTS 7637 05:41:23,920 --> 05:41:27,960 THROUGH THE Q&A BOX. 7638 05:41:27,960 --> 05:41:32,080 THIS IS AN INCREDIBLE INITIATIVE 7639 05:41:32,080 --> 05:41:33,040 BY EVERYONE. 7640 05:41:33,040 --> 05:41:37,160 THERE IS -- EVERYONE IN THE ALS 7641 05:41:37,160 --> 05:41:39,360 STRATEGIC PLANNING COMMITTEE. 7642 05:41:39,360 --> 05:41:41,840 MY HEARTFELT GRATITUDE TO BRING 7643 05:41:41,840 --> 05:41:43,640 HOPE IN THE COMMUNITY. 7644 05:41:43,640 --> 05:41:45,520 DAY BY DAY WE ARE REACHING 7645 05:41:45,520 --> 05:41:47,960 TOWARDS DECODING THIS INCURRABLE 7646 05:41:47,960 --> 05:41:49,640 AND DEVASTATING DISEASE. 7647 05:41:49,640 --> 05:41:56,360 ONE DAY WE WILL SUCCEED. 7648 05:41:56,360 --> 05:41:59,960 THERE WAS ANOTHER ONE BUT IT 7649 05:41:59,960 --> 05:42:05,120 DISAPPEARED FROM MY BOX. 7650 05:42:05,120 --> 05:42:06,280 >>I THINK WE READ ALL THE 7651 05:42:06,280 --> 05:42:07,240 COMMENTS WE RECEIVED. 7652 05:42:07,240 --> 05:42:11,240 WE'RE GRATEFUL FOR ALL OF THE 7653 05:42:11,240 --> 05:42:12,680 COMMENTS THAT EVERYONE POSTED. 7654 05:42:12,680 --> 05:42:14,520 THIS WAS VERY INSPIRING AND 7655 05:42:14,520 --> 05:42:17,840 WE'LL CERTAINLY TAKE THESE 7656 05:42:17,840 --> 05:42:19,400 COMMENTS INTO CONSIDERATION WHEN 7657 05:42:19,400 --> 05:42:22,240 WE REVISIT OUR DRAFT PROPOSAL 7658 05:42:22,240 --> 05:42:23,960 YOU WERE ALL ABLE TO LOOK OVER 7659 05:42:23,960 --> 05:42:24,840 AND ADDRESS. 7660 05:42:24,840 --> 05:42:27,840 I THINK THIS WAS AN INCREDIBLY 7661 05:42:27,840 --> 05:42:28,480 SUCCESSFUL DAY. 7662 05:42:28,480 --> 05:42:30,280 THANK YOU FOR EVERYBODY FOR 7663 05:42:30,280 --> 05:42:31,760 PARTICIPATING. 7664 05:42:31,760 --> 05:42:32,760 THANK YOU TO ALL OF OUR WORKING 7665 05:42:32,760 --> 05:42:34,560 GROUPS AND THEIR MEMBERS FOR 7666 05:42:34,560 --> 05:42:37,040 PUTTING TOGETHER THIS WONDERFUL 7667 05:42:37,040 --> 05:42:38,400 SUMMARY OF HOW THEY PRIORITIZED 7668 05:42:38,400 --> 05:42:40,960 WHERE WE SHOULD GO NEXT AND I 7669 05:42:40,960 --> 05:42:42,960 THINK IT BECAME CLEAR THAT TIME 7670 05:42:42,960 --> 05:42:45,720 IS ONE OF THE THINGS THAT 7671 05:42:45,720 --> 05:42:46,880 ESPECIALLY IN THE COMMENTS 7672 05:42:46,880 --> 05:42:48,080 BECAME OBVIOUS THAT THE ALS 7673 05:42:48,080 --> 05:42:50,320 CLOCK IS AN IMPORTANT ISSUE FOR 7674 05:42:50,320 --> 05:42:54,600 MOST OF THE ALS CAREGIVERS AND 7675 05:42:54,600 --> 05:42:59,840 WE SHOULD KEEP IN MIND MOVING 7676 05:42:59,840 --> 05:43:04,760 FORWARD AND WE'LL DO SO IT'S 7677 05:43:04,760 --> 05:43:07,680 GOOD TO BE REMINDED OF THIS AT 7678 05:43:07,680 --> 05:43:08,960 ALL TIMES AND AN IMPORTANT ISSUE 7679 05:43:08,960 --> 05:43:11,240 THAT'S RELEVANT FOR THE ALS 7680 05:43:11,240 --> 05:43:11,520 COMMUNITY. 7681 05:43:11,520 --> 05:43:13,760 WE APPRECIATE THE HONESTY AND 7682 05:43:13,760 --> 05:43:16,160 COMMENTS IN REGARDS TO MAKING IT 7683 05:43:16,160 --> 05:43:17,560 IMPORTANT FOR US THAT THIS IS 7684 05:43:17,560 --> 05:43:19,400 SOMETHING THAT WE SHOULD ALWAYS 7685 05:43:19,400 --> 05:43:22,440 KEEP IN MIND AS WE MOVE FORWARD. 7686 05:43:22,440 --> 05:43:23,360 BOB, DO YOU WANT A FEW MORE 7687 05:43:23,360 --> 05:43:26,240 COMMENTS BEFORE WE CLOSE UP? 7688 05:43:26,240 --> 05:43:29,280 >>NOT REALLY. 7689 05:43:29,280 --> 05:43:31,000 I THINK IT'S BEEN A MOTIVATING 7690 05:43:31,000 --> 05:43:33,760 AND ALL-IMPORTANT DAY. 7691 05:43:33,760 --> 05:43:37,080 WE SUFFER HISTORICALLY BECAUSE 7692 05:43:37,080 --> 05:43:38,640 ALS IS SAID TO BE AN ORPHAN 7693 05:43:38,640 --> 05:43:40,440 DISEASE AND IF WE LOOK AT THE 7694 05:43:40,440 --> 05:43:42,520 STATISTICS, MORE THAN 500,000 7695 05:43:42,520 --> 05:43:43,440 PEOPLE ALIVE IN THE UNITED 7696 05:43:43,440 --> 05:43:46,240 STATES TODAY WILL DIE OF ALS. 7697 05:43:46,240 --> 05:43:47,960 THAT'S LOOKING AGAIN AND AGAIN 7698 05:43:47,960 --> 05:43:50,760 AT DEATH CERTIFICATES ACROSS THE 7699 05:43:50,760 --> 05:43:53,000 U.S. AND WESTERN EUROPE. 7700 05:43:53,000 --> 05:43:54,520 IT'S NOT AN ORPHAN DISEASE FROM 7701 05:43:54,520 --> 05:43:55,720 THE PERSPECTIVE OF A TOTAL 7702 05:43:55,720 --> 05:43:59,120 BURDEN OF THE MEDICAL CHALLENGE 7703 05:43:59,120 --> 05:44:02,320 AND OF THE TRAGEDY THIS 7704 05:44:02,320 --> 05:44:02,920 REPRESENTS. 7705 05:44:02,920 --> 05:44:07,960 WHILE I'M DEEPLY GRATEFUL TO THE 7706 05:44:07,960 --> 05:44:09,560 NINDS FOR ALL YOU'RE DOING AND 7707 05:44:09,560 --> 05:44:11,120 LOOK FORWARD TO TOMORROW IN 7708 05:44:11,120 --> 05:44:12,720 CONTINUING THE MOTIVATING 7709 05:44:12,720 --> 05:44:13,000 DISCUSSION. 7710 05:44:13,000 --> 05:44:14,000 THANK YOU VERY MUCH. 7711 05:44:14,000 --> 05:44:16,120 >>ALSO, I ECHO WHAT YOU SAID, 7712 05:44:16,120 --> 05:44:16,320 BOB. 7713 05:44:16,320 --> 05:44:19,600 I LOOK FORWARD TO TOMORROW AND 7714 05:44:19,600 --> 05:44:25,600 AS YOU CAN SEE THE COMMUNITY 7715 05:44:25,600 --> 05:44:31,920 INVESTED IN ALS IS WE VOE BUST 7716 05:44:31,920 --> 05:44:34,440 AND INNOVATIVE AND NATION AT AND 7717 05:44:34,440 --> 05:44:36,120 WILLING TO ROLL UP THEIR 7718 05:44:36,120 --> 05:44:36,960 SLEEVES. 7719 05:44:36,960 --> 05:44:38,680 WE'RE ON THE SAME TEAM HERE. 7720 05:44:38,680 --> 05:44:41,480 I WANTED TO ADDRESS ONE THING 7721 05:44:41,480 --> 05:44:43,200 THAT'S ABOUT THE TIME FRAMES. 7722 05:44:43,200 --> 05:44:47,320 THE ACTIONS THAT ARE COMING OUT 7723 05:44:47,320 --> 05:44:49,520 OF THE PLAN CAN INFLUENCE 7724 05:44:49,520 --> 05:44:50,120 RESEARCH STARTING TOMORROW. 7725 05:44:50,120 --> 05:44:52,920 IT'S NOT -- NO ONE HAS TO WAIT. 7726 05:44:52,920 --> 05:44:57,720 THERE'LL BE THINGS THAT COME 7727 05:44:57,720 --> 05:45:00,800 ALONG AT DIFFERENT TIMES BUT THE 7728 05:45:00,800 --> 05:45:01,680 IMPACT SHOULD BE IMMEDIATE. 7729 05:45:01,680 --> 05:45:03,640 WITH THAT I'D LIKE TO THANK 7730 05:45:03,640 --> 05:45:04,960 EVERYONE ONCE AGAIN AND WE'LL 7731 05:45:04,960 --> 05:45:06,920 SEE YOU TOMORROW. 7732 05:45:06,920 --> 05:45:07,440 >>THANK YOU, EVERYBODY. 7733 05:45:07,440 --> 05:45:17,720 SEE YOU TOMORROW.