1 00:00:49,174 --> 00:00:50,942 >> TO THE REGULAR ADMINISTRATIVE 2 00:00:50,942 --> 00:00:51,609 ANNOUNCEMENTS. 3 00:00:51,609 --> 00:00:52,811 WE'RE CURRENTLY AWAITING 4 00:00:52,811 --> 00:00:57,215 DEPARTMENTAL APPROVAL OF OUR 5 00:00:57,215 --> 00:00:58,717 2023 COUNCIL -- FOR ALL OF YOU 6 00:00:58,717 --> 00:01:00,585 ALL TO KNOW AND WE'VE BEGUN THE 7 00:01:00,585 --> 00:01:03,655 PROCESS TO IDENTIFY NOMINEES FOR 8 00:01:03,655 --> 00:01:13,965 THE 2024 VACANCY. 9 00:03:09,097 --> 00:03:13,067 WE FINALLY GOT A BUDGET. 10 00:03:13,067 --> 00:03:17,538 AND OBVIOUSLY YOU'VE SEEN THAT 11 00:03:17,538 --> 00:03:20,108 CURVE GOING UP FOR THE PRECEDING 12 00:03:20,108 --> 00:03:23,044 FIVE YEARS, BUT WE'VE ENTERED 13 00:03:23,044 --> 00:03:24,212 INTO A PHASE WHERE THINGS ARE A 14 00:03:24,212 --> 00:03:29,684 BIT MORE CHALLENGING, AND IN 15 00:03:29,684 --> 00:03:32,320 ESSENCE, WE HAVE A RELATIVELY 16 00:03:32,320 --> 00:03:36,124 FLAT BUDGET FOR THE NIH, AND FOR 17 00:03:36,124 --> 00:03:38,893 NHLBI, AND AS YOU ARE ALL AWARE, 18 00:03:38,893 --> 00:03:41,829 GIVEN THE SORT OF GENERAL 19 00:03:41,829 --> 00:03:42,930 INFLATIONARY PRESSURES ON 20 00:03:42,930 --> 00:03:45,433 EVERYTHING, INCLUDING BIOMEDICAL 21 00:03:45,433 --> 00:03:47,969 SCIENCES THAT YOU'RE ALL AWARE 22 00:03:47,969 --> 00:03:50,371 OF, IN THE CONTEXT OF THAT 23 00:03:50,371 --> 00:03:53,875 INFLATION, A FLAT BUDGET MEANS 24 00:03:53,875 --> 00:03:54,842 THAT INEVITABLY THERE ARE GOING 25 00:03:54,842 --> 00:03:57,445 TO BE CONSTRAINTS AND 26 00:03:57,445 --> 00:03:58,079 TIGHTENING. 27 00:03:58,079 --> 00:04:02,049 SO THIS WILL BE A CHALLENGE FOR 28 00:04:02,049 --> 00:04:04,152 FY 24, AND AT THE BEGINNING OF 29 00:04:04,152 --> 00:04:07,188 THE PROCESS FOR FY 25, THINGS 30 00:04:07,188 --> 00:04:10,024 ARE LOOKING LIKE THE SPACE WILL 31 00:04:10,024 --> 00:04:11,559 BE VERY SIMILAR. 32 00:04:11,559 --> 00:04:13,227 SO WE HAVE A CHALLENGING COUPLE 33 00:04:13,227 --> 00:04:17,064 OF YEARS TO WORK THROUGH. 34 00:04:17,064 --> 00:04:20,668 WE HAD THE PRIVILEGE OF JOINING 35 00:04:20,668 --> 00:04:22,837 THE NIH DIRECTOR AT THE SENATE 36 00:04:22,837 --> 00:04:25,473 APPROPRIATIONS HEARING, WHERE 37 00:04:25,473 --> 00:04:26,240 DR. BERTAGNOLLI OUTLINED A 38 00:04:26,240 --> 00:04:29,410 NUMBER OF PRIORITIES AS WELL AS 39 00:04:29,410 --> 00:04:31,145 WE HEARD FROM OUR APPROPRIATORS 40 00:04:31,145 --> 00:04:34,282 AND SOME OF THE TOPICS MENTIONED 41 00:04:34,282 --> 00:04:36,317 IN THE CONTEXT OF THAT HEARING 42 00:04:36,317 --> 00:04:38,686 ARE ADDRESSED HERE. 43 00:04:38,686 --> 00:04:43,624 RURAL HEALTH, WOMEN'S HEALTH, 44 00:04:43,624 --> 00:04:45,159 MATERNAL MORBIDITY AND 45 00:04:45,159 --> 00:04:46,694 MORTALITY, HEART DISEASE WAS 46 00:04:46,694 --> 00:04:49,530 MENTIONED. 47 00:04:49,530 --> 00:04:51,833 AS WAS A SENSE OF WHAT THESE 48 00:04:51,833 --> 00:04:52,967 AUSTERE BUDGETS MAY HAVE FOR THE 49 00:04:52,967 --> 00:04:54,569 NEXT GENERATION. 50 00:04:54,569 --> 00:04:56,838 BY SOME OF OUR MEMBERS OF 51 00:04:56,838 --> 00:05:00,041 CONGRESS. 52 00:05:00,041 --> 00:05:01,676 SIMILARLY, THERE WAS NOTEWORTHY 53 00:05:01,676 --> 00:05:05,413 QUESTIONS AND DIALOGUE ABOUT 54 00:05:05,413 --> 00:05:07,915 CERTAIN POPULATIONS, TYPICALLY 55 00:05:07,915 --> 00:05:10,318 ASIAN AMERICANS, NATIVE 56 00:05:10,318 --> 00:05:13,488 HAWAIIANS, PACIFIC ISLANDERS, A 57 00:05:13,488 --> 00:05:15,423 LOT OF INTEREST AND EXCITEMENT 58 00:05:15,423 --> 00:05:17,225 AROUND ARTIFICIAL INTELLIGENCE, 59 00:05:17,225 --> 00:05:18,693 MACHINE LEARNING AND ITS 60 00:05:18,693 --> 00:05:20,561 IMPLICATIONS FOR BIOMEDICAL 61 00:05:20,561 --> 00:05:25,700 SCIENCE, AS WELL AS LONG COVID. 62 00:05:25,700 --> 00:05:27,435 SO ALTHOUGH THERE ARE A NUMBER 63 00:05:27,435 --> 00:05:28,769 OF GREAT PRIORITIES THAT ARE 64 00:05:28,769 --> 00:05:29,737 EXCITING, WE'RE GOING TO HAVE TO 65 00:05:29,737 --> 00:05:30,938 WORK THROUGH THAT AT THIS POINT 66 00:05:30,938 --> 00:05:35,776 WITH A RELATIVELY FLAT BUDGET. 67 00:05:35,776 --> 00:05:37,311 THAT OBVIOUSLY HAS IMPLICATIONS 68 00:05:37,311 --> 00:05:39,247 ON HOW WE'VE BEEN ABLE TO 69 00:05:39,247 --> 00:05:41,649 NAVIGATE THIS. 70 00:05:41,649 --> 00:05:46,587 WE'VE TRIED TO MAINTAIN THOSE 71 00:05:46,587 --> 00:05:48,756 OPERATING PRINCIPLES, 72 00:05:48,756 --> 00:05:51,726 RECOGNIZING THAT PROBABLY THE 73 00:05:51,726 --> 00:05:53,694 GROUP THAT PAYS THE GREATEST 74 00:05:53,694 --> 00:05:57,865 COST IN THESE AUSTERE TIMES 75 00:05:57,865 --> 00:06:02,169 TENDS TO BE OUR NEXT GENERATION. 76 00:06:02,169 --> 00:06:03,671 IT'S ALWAYS HARD TO GET A 77 00:06:03,671 --> 00:06:05,172 FOOTHOLD WHEN THE PAYLINES ARE 78 00:06:05,172 --> 00:06:06,607 FALLING. 79 00:06:06,607 --> 00:06:07,808 THE SENIOR INVESTIGATORS ARE 80 00:06:07,808 --> 00:06:10,578 ABLE TO ADAPT, HAVE MORE GRANTS, 81 00:06:10,578 --> 00:06:12,613 ET CETERA, SO WE'VE TRIED TO 82 00:06:12,613 --> 00:06:17,985 STRIVE TO MAINTAIN THOSE 83 00:06:17,985 --> 00:06:19,320 PAYLINES. 84 00:06:19,320 --> 00:06:21,489 THAT HAS MEANT THAT WE DID 85 00:06:21,489 --> 00:06:23,524 SOMETHING WE TYPICALLY DON'T DO 86 00:06:23,524 --> 00:06:28,362 AT THE NHLBI, DONE AT OTHER 87 00:06:28,362 --> 00:06:31,332 INSTITUTES, WHERE WE GIVE A HAIR 88 00:06:31,332 --> 00:06:33,935 CUT, A 2% REDUCTION IN THE 89 00:06:33,935 --> 00:06:35,169 NONCOMPETING AWARDS, SINCE WHAT 90 00:06:35,169 --> 00:06:39,307 WE HAD TO DO THIS YEAR, IN ORDER 91 00:06:39,307 --> 00:06:42,910 TO HAVE THE CORPUS TO TRY TO 92 00:06:42,910 --> 00:06:45,980 MAINTAIN AT LEAST OUR 14TH 93 00:06:45,980 --> 00:06:47,448 PERCENTILE. 94 00:06:47,448 --> 00:06:49,150 IF WE HAD NOT DONE THAT, 95 00:06:49,150 --> 00:06:50,785 OBVIOUSLY THIS WOULD HAVE BEEN 96 00:06:50,785 --> 00:06:53,087 COMPROMISED IN TERMS OF NUMBER 97 00:06:53,087 --> 00:06:55,823 OF R01s AND SUCCESS RATES 98 00:06:55,823 --> 00:06:57,158 WE'RE ABLE TO MAINTAIN. 99 00:06:57,158 --> 00:06:58,459 SO YOU CAN SEE THERE, DESPITE 100 00:06:58,459 --> 00:06:59,660 THE FACT THAT WE HAVE ACTUALLY 101 00:06:59,660 --> 00:07:05,132 MORE APPLICATIONS COMING IN, 102 00:07:05,132 --> 00:07:07,635 WITH THAT HAIR CUT ACROSS THE 103 00:07:07,635 --> 00:07:09,403 BOARD, THAT HAS ENABLED US TO 104 00:07:09,403 --> 00:07:12,573 TRY TO MAINTAIN AT LEAST HEAD 105 00:07:12,573 --> 00:07:13,874 ABOVE WATER. 106 00:07:13,874 --> 00:07:15,409 AND WE'RE HOPEFUL THAT WE CAN 107 00:07:15,409 --> 00:07:18,579 STILL MAINTAIN OUR TARGET FOR 108 00:07:18,579 --> 00:07:19,513 EARLY STAGE INVESTIGATORS AS 109 00:07:19,513 --> 00:07:21,549 PART OF THAT. 110 00:07:21,549 --> 00:07:24,485 WE'VE ALSO TRIED TO MAINTAIN OUR 111 00:07:24,485 --> 00:07:25,920 TARGETS FOR CAREER DEVELOPMENT 112 00:07:25,920 --> 00:07:27,989 AWARDS AND OTHER ELEMENTS 113 00:07:27,989 --> 00:07:30,825 RELATED TO TRAINEES. 114 00:07:30,825 --> 00:07:32,793 IN ADDITION, AS YOU'RE PROBABLY 115 00:07:32,793 --> 00:07:35,963 AWARE, WE DID I THINK ABOUT AN 116 00:07:35,963 --> 00:07:37,932 8% INCREASE, FAIRLY SUBSTANTIAL 117 00:07:37,932 --> 00:07:41,769 JUMP FOR ONE YEAR OF OUR 118 00:07:41,769 --> 00:07:43,838 STIPENDS AND SO FORTH FOR 119 00:07:43,838 --> 00:07:45,806 POSTDOCS. 120 00:07:45,806 --> 00:07:48,542 SO DESPITE THE AUSTERE BUDGET, 121 00:07:48,542 --> 00:07:54,015 WE'VE TRIED TO FOCUS ON THE NEXT 122 00:07:54,015 --> 00:07:56,651 GENERATION, ENSURING THAT THEY 123 00:07:56,651 --> 00:07:59,820 KIND OF GET THROUGH THIS AND 124 00:07:59,820 --> 00:08:01,555 WEATHER THIS PERIOD OF TIME. 125 00:08:01,555 --> 00:08:05,292 BUT IT DOES CAUSE CONSTRAINTS 126 00:08:05,292 --> 00:08:09,130 THAT WILL HAVE IMPLICATIONS. 127 00:08:09,130 --> 00:08:11,966 TO SOME MORE POSITIVE NEWS, JUST 128 00:08:11,966 --> 00:08:16,003 WANT TO GIVE YOU A SENSE OF SOME 129 00:08:16,003 --> 00:08:18,072 OF THE TRANSITIONS IN LEADERSHIP 130 00:08:18,072 --> 00:08:21,075 THAT WE HAVE, THIS IS ALSO VERY 131 00:08:21,075 --> 00:08:24,245 EXCITING BECAUSE THIS IS AN 132 00:08:24,245 --> 00:08:25,513 OPPORTUNITY FOR THE INSTITUTION, 133 00:08:25,513 --> 00:08:27,281 THE ORGANIZATION TO CONTINUE TO 134 00:08:27,281 --> 00:08:32,520 RENEW ITSELF AND ACTUALLY HAVE A 135 00:08:32,520 --> 00:08:33,721 NEW GENERATION OF LEADERS COMING 136 00:08:33,721 --> 00:08:35,156 THROUGH, WHICH IS EXCITING, AND 137 00:08:35,156 --> 00:08:37,858 HOPEFULLY YOU CAN SEE THE 138 00:08:37,858 --> 00:08:39,393 INCLUSIVE EXCELLENCE OF THESE 139 00:08:39,393 --> 00:08:41,262 FOLKS AS THEY TRANSITION INTO 140 00:08:41,262 --> 00:08:48,369 LEADERSHIP ROLES. 141 00:08:48,369 --> 00:08:55,609 TRACI IN DBDR, MICHELLE IN DCDS 142 00:08:55,609 --> 00:08:58,646 AND COURTNEY ALSO IN DBDR. 143 00:08:58,646 --> 00:09:01,048 SO WE'RE EXCITED TO HAVE THEM AS 144 00:09:01,048 --> 00:09:02,249 NEW LEADERS. 145 00:09:02,249 --> 00:09:05,553 SIMILARLY, WE'RE ALWAYS EXCITED, 146 00:09:05,553 --> 00:09:08,956 JIM WILL TELL YOU THAT THE LUNG 147 00:09:08,956 --> 00:09:11,025 DIVISION IS THE GREATEST 148 00:09:11,025 --> 00:09:14,095 DIVISION IN THE NHLBI. 149 00:09:14,095 --> 00:09:18,466 HE SAYS THAT UNABASHEDLY, I SEE 150 00:09:18,466 --> 00:09:21,836 HIM SMILING BACK THERE, AND HE'S 151 00:09:21,836 --> 00:09:24,572 LED AN ADAPTIVE CHANGE IN 152 00:09:24,572 --> 00:09:28,676 PURSUIT INVOLVING SCIENTIFIC 153 00:09:28,676 --> 00:09:31,779 OPPORTUNITIES, MOVE FROM THE 154 00:09:31,779 --> 00:09:33,781 TWO-BRANCH STRUCTURE TO ONE THAT 155 00:09:33,781 --> 00:09:37,718 DLD BELIEVES IS CAPTURING THE 156 00:09:37,718 --> 00:09:39,353 SPECTRUM OF SCIENCE. 157 00:09:39,353 --> 00:09:44,492 DLD HAS ALSO EXPANDED UNDER HIS 158 00:09:44,492 --> 00:09:45,493 LEADERSHIP, AND I THINK THIS 159 00:09:45,493 --> 00:09:49,296 PUTS DLD IN A VERY GOOD POSITION 160 00:09:49,296 --> 00:09:50,598 TO ALIGN THE EXPERTISE AND 161 00:09:50,598 --> 00:09:52,366 CAPABILITIES AND REALLY THE 162 00:09:52,366 --> 00:09:53,334 SCIENTIFIC OPPORTUNITIES THAT 163 00:09:53,334 --> 00:09:55,536 ARE EMERGING IN LUNG DISEASE 164 00:09:55,536 --> 00:09:58,939 THAT WE'LL TOUCH ON AS WELL. 165 00:09:58,939 --> 00:09:59,907 SIMILARLY, IT GIVES AN 166 00:09:59,907 --> 00:10:02,543 OPPORTUNITY FOR ALL THOSE YOUNG 167 00:10:02,543 --> 00:10:04,612 LEADERS TO EMERGE AND HELP US 168 00:10:04,612 --> 00:10:12,052 DRIVE THE ORGANIZATION. 169 00:10:12,052 --> 00:10:13,154 TOWARD THAT END, THIS IS 170 00:10:13,154 --> 00:10:16,323 PROBABLY A LITTLE OF A PREVIEW, 171 00:10:16,323 --> 00:10:22,096 BUT COMING ATTRACTIONS, NEW 172 00:10:22,096 --> 00:10:23,531 DIVISION LEADERSHIP ALSO IN DLD 173 00:10:23,531 --> 00:10:25,266 IS EMERGING, AND THERE'S SOME 174 00:10:25,266 --> 00:10:25,733 FINALISTS HERE. 175 00:10:25,733 --> 00:10:30,304 SO WE'RE VERY EXCITED ABOUT THE 176 00:10:30,304 --> 00:10:36,243 DIRECTION OF OUR LUNG DIVISION. 177 00:10:36,243 --> 00:10:41,482 I'D BE REMISS IF I ALSO DIDN'T 178 00:10:41,482 --> 00:10:44,218 ACKNOWLEDGE THE SUPERLATIVE 179 00:10:44,218 --> 00:10:45,286 LEADERSHIP THE DIVISION OF LUNG 180 00:10:45,286 --> 00:10:47,822 DISEASE HAS HAD, IN PARTICULAR, 181 00:10:47,822 --> 00:10:50,758 A MILESTONE. 182 00:10:50,758 --> 00:10:54,395 IT WAS 1984 WHEN DR. KILEY 183 00:10:54,395 --> 00:10:57,264 JOINED THE ORGANIZATION AS A 184 00:10:57,264 --> 00:10:57,665 YOUNG PUP. 185 00:10:57,665 --> 00:11:07,708 I WON'T SAY HOW YOUNG HE WAS. 186 00:11:07,708 --> 00:11:09,376 AND HAS REALLY BLAZED A PATHWAY 187 00:11:09,376 --> 00:11:16,150 AS A PROGRAM OFFICER, WHO WAS 188 00:11:16,150 --> 00:11:20,287 SEEN AS AN EMERGING STAR, 189 00:11:20,287 --> 00:11:20,955 INCREASING OPPORTUNITIES OF 190 00:11:20,955 --> 00:11:25,659 LEADERSHIP TO THE POINT WHERE HE 191 00:11:25,659 --> 00:11:26,760 BECAME DIRECTOR OF DIVISION OF 192 00:11:26,760 --> 00:11:28,495 LUNG DISEASES. 193 00:11:28,495 --> 00:11:32,333 AND HE'S LED A VARIETY OF 194 00:11:32,333 --> 00:11:33,434 PROGRAMS, I CAN'T EVEN LIST ALL 195 00:11:33,434 --> 00:11:36,170 THE THINGS THAT DR. KILEY HAS 196 00:11:36,170 --> 00:11:39,006 DONE OVER HIS 40 YEARS OF 197 00:11:39,006 --> 00:11:39,240 SERVICE. 198 00:11:39,240 --> 00:11:40,741 DOING A LOT OF THE PIONEERING 199 00:11:40,741 --> 00:11:42,176 WORK WITH DEVELOPING CLINICAL 200 00:11:42,176 --> 00:11:49,717 TRIAL NETWORKS, HERE'S JUST 201 00:11:49,717 --> 00:11:51,585 LISTING CADET, THE THINGS 202 00:11:51,585 --> 00:11:52,887 THEY'VE DONE, OXYGEN, HE 203 00:11:52,887 --> 00:11:54,922 ACTUALLY WAS THE FIRST DIRECTOR 204 00:11:54,922 --> 00:11:57,391 OF THE NATIONAL CENTER ON SLEEP 205 00:11:57,391 --> 00:12:01,195 DISORDER RESEARCH, AND SO I JUST 206 00:12:01,195 --> 00:12:03,831 WANTED TO TAKE A MOMENT TO JUST 207 00:12:03,831 --> 00:12:07,001 GIVE A VERY SPECIAL SHOUT OUT TO 208 00:12:07,001 --> 00:12:10,437 A DEDICATED SERVANT. 209 00:12:10,437 --> 00:12:14,341 JIM AND I SOMETIMES GET INTO 210 00:12:14,341 --> 00:12:19,146 DEBATES IN OUR EXECUTIVE 211 00:12:19,146 --> 00:12:20,481 LEADERSHIP TEAM MEETINGS, AND I 212 00:12:20,481 --> 00:12:23,183 LOVE HIS PERSPECTIVE. 213 00:12:23,183 --> 00:12:25,486 HE HAS A LOT OF HISTORY AND 214 00:12:25,486 --> 00:12:26,587 EXPERIENCE, SO I HAVE GREAT 215 00:12:26,587 --> 00:12:30,624 RESPECT FOR THAT PERSPECTIVE, AS 216 00:12:30,624 --> 00:12:31,825 HOPEFULLY MY EXECUTIVE TEAM 217 00:12:31,825 --> 00:12:33,027 WOULD ACKNOWLEDGE. 218 00:12:33,027 --> 00:12:35,429 I LIKE THE BACK AND FORTH, THE 219 00:12:35,429 --> 00:12:35,763 DIALOGUE. 220 00:12:35,763 --> 00:12:37,398 I THINK THAT MAKES US BETTER AS 221 00:12:37,398 --> 00:12:41,802 AN ORGANIZATION. 222 00:12:41,802 --> 00:12:44,972 AND ALTHOUGH WE MAY NOT ALWAYS 223 00:12:44,972 --> 00:12:46,473 AGREE ON TACTICAL APPROACHES, 224 00:12:46,473 --> 00:12:51,412 ONE THING I DO KNOW ABOUT JIM 225 00:12:51,412 --> 00:12:52,646 KILEY IS THAT HE WOULD DO 226 00:12:52,646 --> 00:12:54,281 ANYTHING IF HE THINKS IT'S THE 227 00:12:54,281 --> 00:12:58,185 BEST FOR THE INSTITUTE, AND IN 228 00:12:58,185 --> 00:12:58,852 FULFILLING OUR MISSION. 229 00:12:58,852 --> 00:13:02,890 SO I HAVE NO DOUBT ABOUT HIS 230 00:13:02,890 --> 00:13:05,092 DEDICATION TO THE NHLBI AND ALL 231 00:13:05,092 --> 00:13:07,695 THAT IT DOES. 232 00:13:07,695 --> 00:13:09,563 SO JIM, WE JUST WANT TO 233 00:13:09,563 --> 00:13:10,664 ACKNOWLEDGE YOU IN YOUR 40 YEARS 234 00:13:10,664 --> 00:13:11,665 OF SERVICE. 235 00:13:11,665 --> 00:13:21,842 [APPLAUSE] 236 00:13:29,750 --> 00:13:31,218 I CAN TELL YOU I PROBABLY WON'T 237 00:13:31,218 --> 00:13:32,987 MAKE TO THE 40-YEAR MARK, THAT'S 238 00:13:32,987 --> 00:13:33,754 FOR SURE. 239 00:13:33,754 --> 00:13:38,892 SO WE THANK JIM FOR THAT. 240 00:13:38,892 --> 00:13:42,062 I ALSO WANTED TO GIVE AN 241 00:13:42,062 --> 00:13:45,466 ACKNOWLEDGMENT TO ANOTHER PART 242 00:13:45,466 --> 00:13:55,409 OF OUR EXECUTIVE LEADERSHIP, DRW 243 00:13:55,409 --> 00:13:58,912 SCIENTIFIC DIRECTOR OF OUR 244 00:13:58,912 --> 00:13:59,680 INTRAMURAL PROGRAM, 245 00:13:59,680 --> 00:14:00,481 PARTICIPATING IN THE OVERSIGHT 246 00:14:00,481 --> 00:14:02,616 OF OUR BOARD OF SCIENTIFIC 247 00:14:02,616 --> 00:14:04,685 COUNSELOR MEETING AND 248 00:14:04,685 --> 00:14:05,886 EVALUATIONS, THAT WAS PART OF 249 00:14:05,886 --> 00:14:07,087 THE CLOSED SESSION. 250 00:14:07,087 --> 00:14:10,991 AND SO WE'RE DELIGHTED TO HAVE 251 00:14:10,991 --> 00:14:15,863 DR. CHILDS AS THE LEADER OF THE 252 00:14:15,863 --> 00:14:16,964 INTRAMURAL PROGRAM, EXCITED 253 00:14:16,964 --> 00:14:18,932 ABOUT HIS GREAT VISION, HAVING 254 00:14:18,932 --> 00:14:21,301 BEEN THE DIRECTOR -- THE 255 00:14:21,301 --> 00:14:23,404 CLINICAL DIRECTOR OF THE DIR, 256 00:14:23,404 --> 00:14:30,110 NOW TAKING THE HELM FROM BOB 257 00:14:30,110 --> 00:14:32,579 BALABAN AFTER BOB'S 25 YEARS AS 258 00:14:32,579 --> 00:14:35,215 SD, SO WE'RE VERY EXCITED ABOUT 259 00:14:35,215 --> 00:14:38,285 THE DIRECTION OF THE INTRAMURAL 260 00:14:38,285 --> 00:14:43,957 PROGRAM IN THE NHLBI. 261 00:14:43,957 --> 00:14:47,561 A KEY PART OF DR. CHILDS, QUITE 262 00:14:47,561 --> 00:14:48,562 FRANKLY, RESPONSIBILITY AND 263 00:14:48,562 --> 00:14:50,964 STEWARDSHIP IS, AGAIN, FOR THE 264 00:14:50,964 --> 00:14:53,133 NEXT GENERATION, AND WE'RE VERY 265 00:14:53,133 --> 00:14:55,235 EXCITED ABOUT THIS INCREDIBLE 266 00:14:55,235 --> 00:14:58,072 GROUP OF TENURED TRACKERS THAT 267 00:14:58,072 --> 00:15:00,374 HE WILL BE MENTORING AND 268 00:15:00,374 --> 00:15:01,909 SHEPHERDING IN THEIR CAREER 269 00:15:01,909 --> 00:15:02,876 TRAJECTORY. 270 00:15:02,876 --> 00:15:05,112 AND WE'RE DELIGHTED THAT TODAY 271 00:15:05,112 --> 00:15:07,815 AS PART OF OUR OVERALL VIEW OF 272 00:15:07,815 --> 00:15:11,218 THE INTRAMURAL PROGRAM, WE'LL 273 00:15:11,218 --> 00:15:14,788 HAVE DR. COURTNEY FITZHUGH WHO 274 00:15:14,788 --> 00:15:16,190 WILL BE PRESENTING SOME OF HER 275 00:15:16,190 --> 00:15:18,859 WORK TO GIVE YOU A GLIMPSE OF 276 00:15:18,859 --> 00:15:19,927 SOME OF THE SCIENCE THAT'S BEING 277 00:15:19,927 --> 00:15:21,495 DONE IN THE NHLBI INTRAMURAL 278 00:15:21,495 --> 00:15:26,066 PROGRAM. 279 00:15:26,066 --> 00:15:28,702 SO LET ME PIVOT FROM THE 280 00:15:28,702 --> 00:15:29,903 STEWARDSHIP TO WHERE WE'RE GOING 281 00:15:29,903 --> 00:15:32,072 IN TERMS OF OUR SCIENTIFIC 282 00:15:32,072 --> 00:15:34,374 OPPORTUNITIES, OF WHICH THE 283 00:15:34,374 --> 00:15:36,243 FIRST PART RELATES TO THE 284 00:15:36,243 --> 00:15:38,879 STRATEGIC VISION REFRESH THAT 285 00:15:38,879 --> 00:15:41,815 BOTH COUNCIL AND BOARD OF 286 00:15:41,815 --> 00:15:42,816 EXTERNAL EXPERTS HAS BEEN 287 00:15:42,816 --> 00:15:44,985 INSTRUMENTAL IN HELPING US TO 288 00:15:44,985 --> 00:15:50,224 DEVELOP AND SHAPE SINCE THE LAST 289 00:15:50,224 --> 00:15:53,627 STRATEGIC VISION, AND THIS IS AN 290 00:15:53,627 --> 00:15:56,396 OPPORTUNITY FOR US TO HEAR FROM 291 00:15:56,396 --> 00:15:58,565 THE COMMUNITY, GET YOUR EXPERT 292 00:15:58,565 --> 00:16:01,768 INPUT AS WE SHAPE THE DIRECTION 293 00:16:01,768 --> 00:16:05,606 FOR THE NEXT FIVE TO 10 YEARS. 294 00:16:05,606 --> 00:16:07,407 AND TOWARD THAT END, WE'RE 295 00:16:07,407 --> 00:16:09,610 MAKING PROGRESS. 296 00:16:09,610 --> 00:16:14,948 THANKS TO DR. LAURA JOHNSON AND 297 00:16:14,948 --> 00:16:19,820 OUR OSPEAK GROUP ALONG WITH 298 00:16:19,820 --> 00:16:21,321 PROGRAM STAFF ARE WORKING 299 00:16:21,321 --> 00:16:23,590 DILIGENTLY TO DO THE OUTREACH TO 300 00:16:23,590 --> 00:16:27,094 THE COMMUNITY TO START TO GET A 301 00:16:27,094 --> 00:16:31,798 SENSE OF IDEAS THAT ARE BUBBLING 302 00:16:31,798 --> 00:16:33,433 UP, STRATEGIC DIRECTIONS WE 303 00:16:33,433 --> 00:16:34,768 MIGHT TAKE OVER THE NEXT FIVE TO 304 00:16:34,768 --> 00:16:36,637 10 YEARS OF THIS REFRESH, SO 305 00:16:36,637 --> 00:16:38,372 LOOKING AT OUR COMPELLING 306 00:16:38,372 --> 00:16:39,473 QUESTIONS AND CRITICAL 307 00:16:39,473 --> 00:16:41,008 CHALLENGES TO SEE IF THOSE HAVE 308 00:16:41,008 --> 00:16:45,379 CHANGED OR EVOLVED DURING THIS 309 00:16:45,379 --> 00:16:46,880 PERIOD, AND NOTABLY THIS IS SOME 310 00:16:46,880 --> 00:16:49,850 OF THE PROGRESS THAT'S BEEN MADE 311 00:16:49,850 --> 00:16:51,718 IN THAT PROCESS. 312 00:16:51,718 --> 00:16:53,120 STAFF HAS DONE AN INCREDIBLE 313 00:16:53,120 --> 00:16:55,222 AMOUNT OF WORK TO KIND OF REVIEW 314 00:16:55,222 --> 00:16:58,058 ALL THOSE, AND IT'S BECOMING 315 00:16:58,058 --> 00:17:00,794 NOTABLE THAT THERE'S CERTAIN 316 00:17:00,794 --> 00:17:04,731 THEMES THAT APPEAR TO BE 317 00:17:04,731 --> 00:17:05,065 EMERGING. 318 00:17:05,065 --> 00:17:07,801 SOME OF IT RELATES TO 319 00:17:07,801 --> 00:17:09,803 OPPORTUNITIES IN WOMEN'S HEALTH 320 00:17:09,803 --> 00:17:11,505 IN PARTICULAR SEEMS TO COME UP. 321 00:17:11,505 --> 00:17:15,209 OBVIOUSLY DATA SCIENCE AND AI/ML 322 00:17:15,209 --> 00:17:17,211 BEING A KEY ONE, BUT ALSO OTHER 323 00:17:17,211 --> 00:17:19,813 AREAS OF IMPLEMENTATION SCIENCE, 324 00:17:19,813 --> 00:17:24,952 SOCIAL DETERMINANT OF HEALTH, 325 00:17:24,952 --> 00:17:28,555 THE MOTION OF MULTILEVEL 326 00:17:28,555 --> 00:17:29,489 INTERVENTIONS, ACTUALLY 327 00:17:29,489 --> 00:17:30,991 INTERSECTIONS LIKE WITH HIV. 328 00:17:30,991 --> 00:17:34,027 SO WE'RE VERY INTRIGUED BY THAT 329 00:17:34,027 --> 00:17:38,298 REQUEST FOR INFORMATION AND A 330 00:17:38,298 --> 00:17:42,035 LOT OF THINGS THAT CAME FROM THE 331 00:17:42,035 --> 00:17:43,003 INTRAMURAL COMMUNITY ABOUT OUR 332 00:17:43,003 --> 00:17:46,440 FUTURE DIRECTIONS. 333 00:17:46,440 --> 00:17:49,676 NOW, JUST AS PART OF THAT 334 00:17:49,676 --> 00:17:52,546 STRATEGIC VISION REFRESH, ONE OF 335 00:17:52,546 --> 00:17:54,181 THE THINGS THAT WE'LL JUST TOUCH 336 00:17:54,181 --> 00:17:57,084 UPON ARE A COUPLE THEMATIC 337 00:17:57,084 --> 00:17:58,552 OPPORTUNITIES THAT ALIGN WITH A 338 00:17:58,552 --> 00:18:03,790 LOT OF THE INPUT WE RECEIVE THAT 339 00:18:03,790 --> 00:18:05,592 WE HOPE CAN CONTINUE TO LEVERAGE 340 00:18:05,592 --> 00:18:07,628 THIS VIRTUAL CYCLE OF DISCOVERY 341 00:18:07,628 --> 00:18:10,130 SCIENCE THAT WE HOPE TO 342 00:18:10,130 --> 00:18:11,665 TRANSLATE INTO THE HEALTH OF ALL 343 00:18:11,665 --> 00:18:13,967 COMMUNITIES. 344 00:18:13,967 --> 00:18:15,369 AND WANTED TO GIVE A PARTICULAR 345 00:18:15,369 --> 00:18:17,904 SHOUT OUT TO BLOOD DIVISION IN 346 00:18:17,904 --> 00:18:22,175 THIS REGARD, AS WE'RE EXCITED 347 00:18:22,175 --> 00:18:26,113 ABOUT THE TWO FDA-APPROVED GENE 348 00:18:26,113 --> 00:18:28,282 THERAPY FOR SICKLE CELL DISEASE. 349 00:18:28,282 --> 00:18:31,351 IT CAME THIS YEAR AS A MAJOR 350 00:18:31,351 --> 00:18:33,086 MILESTONE THAT I THINK REFLECTS 351 00:18:33,086 --> 00:18:39,459 A LOT OF THE INSTITUTE'S 352 00:18:39,459 --> 00:18:43,697 PORTFOLIO, PARTICULARLY IN THE 353 00:18:43,697 --> 00:18:45,032 BLOOD DIVISION THAT SPANS A 354 00:18:45,032 --> 00:18:47,668 BASIC FIRST IN HUMAN CLINICAL 355 00:18:47,668 --> 00:18:49,736 TRANSLATION, AS WELL AS 356 00:18:49,736 --> 00:18:53,874 IMPLEMENTATION SCIENCE NA WE 357 00:18:53,874 --> 00:18:57,110 BELIEVE HAS MADE A DRAMATIC 358 00:18:57,110 --> 00:18:59,146 IMPACT, TRANSFORMATIVE IMPACT ON 359 00:18:59,146 --> 00:19:01,748 SOME OF THE PATIENTS RECEIVING 360 00:19:01,748 --> 00:19:02,416 THESE THERAPIES. 361 00:19:02,416 --> 00:19:03,917 A PICTURE OF JANELLE DOWN THERE 362 00:19:03,917 --> 00:19:06,353 IN THE CORNER IN WHICH IN MANY 363 00:19:06,353 --> 00:19:09,089 WAYS EMBODIES THE PROUD LEGACY 364 00:19:09,089 --> 00:19:11,058 OF THE NHLBI IN MAKING A 365 00:19:11,058 --> 00:19:14,895 DIFFERENCE FOR RARE DISEASES. 366 00:19:14,895 --> 00:19:20,567 IT'S ALSO ONE WHERE IT'S BEEN A 367 00:19:20,567 --> 00:19:22,202 KEY PART OF COLLABORATIVE 368 00:19:22,202 --> 00:19:22,836 PARTNERSHIPS WITH PATIENTS AT 369 00:19:22,836 --> 00:19:27,908 THE CENTER. 370 00:19:27,908 --> 00:19:30,077 AS WELL AS SISTER AGENCIES LIKE 371 00:19:30,077 --> 00:19:33,380 FDA AND CMS TO MAKE SURE THAT 372 00:19:33,380 --> 00:19:35,115 THESE CURATIVE THERAPIES AND 373 00:19:35,115 --> 00:19:36,316 DRAMATIC TRANSFORMATIONAL 374 00:19:36,316 --> 00:19:38,185 TECHNOLOGIES ACTUALLY MEET 375 00:19:38,185 --> 00:19:40,487 PATIENTS WHERE THEY ARE AND ARE 376 00:19:40,487 --> 00:19:47,694 ACCESSIBLE TO ALL OF THEM. 377 00:19:47,694 --> 00:19:50,864 INDEED THAT ABILITY TO TRANSLATE 378 00:19:50,864 --> 00:19:54,901 DISCOVERY SCIENCE IN A WAY THAT 379 00:19:54,901 --> 00:19:56,837 DISSEMINATES AND IS TAKEN UP AT 380 00:19:56,837 --> 00:19:58,638 SCALE OFTEN INVOLVES 381 00:19:58,638 --> 00:20:00,507 COMMERCIALIZATION IN OUR FREE 382 00:20:00,507 --> 00:20:01,375 MARKET SOCIETY. 383 00:20:01,375 --> 00:20:02,909 AND SO WE WANT TO BE SURE THAT 384 00:20:02,909 --> 00:20:07,814 WE CONTINUE THAT ENGINE OF 385 00:20:07,814 --> 00:20:09,583 ACCELERATED TRANSLATION, AND AS 386 00:20:09,583 --> 00:20:13,954 PART OF COUNCIL'S DELIBERATIONS, 387 00:20:13,954 --> 00:20:17,224 WE'RE EXCITED ABOUT THE RENEWAL 388 00:20:17,224 --> 00:20:20,293 OF THE CATALYZE SUITE OF 389 00:20:20,293 --> 00:20:23,029 INITIATIVES THAT'S ALL ABOUT 390 00:20:23,029 --> 00:20:24,798 THAT TRANSLATIONAL SPACE AND 391 00:20:24,798 --> 00:20:26,299 ENABLING THE SUCCESS AND IDEAS 392 00:20:26,299 --> 00:20:28,835 OF INVESTIGATORS LIKE 393 00:20:28,835 --> 00:20:34,508 YOURSELVES. 394 00:20:34,508 --> 00:20:35,809 ALSO WANTED TO, IN THAT SAME 395 00:20:35,809 --> 00:20:38,345 WAY, GIVE A SHOUT OUT TO THE 396 00:20:38,345 --> 00:20:39,546 CURE SICKLE CELL INITIATIVE, 397 00:20:39,546 --> 00:20:40,947 WHICH IS AGAIN SORT OF 398 00:20:40,947 --> 00:20:43,049 EMBLEMATIC OF WHAT WE THINK IS 399 00:20:43,049 --> 00:20:44,217 GOING TO CONTINUE OVER THE NEXT 400 00:20:44,217 --> 00:20:50,590 FIVE TO 10 YEARS WITH THESE NEW 401 00:20:50,590 --> 00:20:53,226 TOOLS AND TECHNOLOGIES, SIMILAR 402 00:20:53,226 --> 00:20:54,728 ACTUALLY TO WHAT WAS USED IN THE 403 00:20:54,728 --> 00:20:57,063 VACCINES AND OTHER FORMS OF IN 404 00:20:57,063 --> 00:20:59,900 VIVO GENE TRANSFER ARE, WE 405 00:20:59,900 --> 00:21:05,138 THINK, GOING TO GO ON AN 406 00:21:05,138 --> 00:21:06,440 EXPONENTIAL OR INFLECTION AT 407 00:21:06,440 --> 00:21:07,808 LEAST IN THE NEXT FIVE TO 10 408 00:21:07,808 --> 00:21:08,542 YEARS. 409 00:21:08,542 --> 00:21:11,745 IN MANY WAYS, THE SICKLE CELL 410 00:21:11,745 --> 00:21:15,982 INITIATIVE IS PRESUMABLY A 411 00:21:15,982 --> 00:21:17,384 FORELONGER OF THAT AND IS IN 412 00:21:17,384 --> 00:21:20,220 MANY WAYS INVESTMENTS IN BASIC 413 00:21:20,220 --> 00:21:22,756 SCIENCE, DISCOVERY SCIENCE TO 414 00:21:22,756 --> 00:21:27,461 APPRECIATE STRATEGIES BY WHICH 415 00:21:27,461 --> 00:21:30,997 WE COULD CURE SICKLE CELL 416 00:21:30,997 --> 00:21:33,033 DISEASE, AND APPLYING THOSE 417 00:21:33,033 --> 00:21:33,467 TECHNOLOGIES. 418 00:21:33,467 --> 00:21:34,434 FURTHER TECHNOLOGY DEVELOPMENT 419 00:21:34,434 --> 00:21:36,536 IS GOING TO BE NECESSARY TO SEE 420 00:21:36,536 --> 00:21:39,372 IF WE COULD DO THIS IN VIVO AND 421 00:21:39,372 --> 00:21:41,775 LEAPFROG TECHNOLOGIES TO TURN 422 00:21:41,775 --> 00:21:43,643 THAT EX VIVO BONE MARROW 423 00:21:43,643 --> 00:21:45,178 TRANSPLANT DRIVEN APPROACH TO 424 00:21:45,178 --> 00:21:48,682 SOMETHING THAT IS CLOSER TO A 425 00:21:48,682 --> 00:21:49,216 BLOOD TRANSFUSION. 426 00:21:49,216 --> 00:21:54,788 SO THAT'S THE NEXT TARGET TO 427 00:21:54,788 --> 00:21:55,355 CURE SICKLE CELL. 428 00:21:55,355 --> 00:21:56,723 I JUST WANTED TO GIVE A SHOUT 429 00:21:56,723 --> 00:21:58,291 OUT AGAIN TO THAT ECOSYSTEM 430 00:21:58,291 --> 00:22:01,561 THAT'S EVOLVED IN THE PRIVATE 431 00:22:01,561 --> 00:22:02,462 SECTOR IN THIS SPACE. 432 00:22:02,462 --> 00:22:08,902 THERE'S ALSO A NICE INTERSECTION 433 00:22:08,902 --> 00:22:12,072 APROPOS TO TODAY WHERE WE SEE 434 00:22:12,072 --> 00:22:16,309 THE COMPLEMENTARITY BETWEEN THE 435 00:22:16,309 --> 00:22:17,344 INTRAMURAL AND EXTRAMURAL 436 00:22:17,344 --> 00:22:18,011 PROGRAM. 437 00:22:18,011 --> 00:22:22,415 HOPEFULLY SOME OF YOU ARE AWARE, 438 00:22:22,415 --> 00:22:23,550 OUR BRANCH CHIEF IN THE 439 00:22:23,550 --> 00:22:25,118 INTRAMURAL PROGRAM IS AN AWARDEE 440 00:22:25,118 --> 00:22:28,588 OF THE SHAW PRIZE, INCREDIBLY 441 00:22:28,588 --> 00:22:32,859 PRESTIGIOUS AWARD. 442 00:22:32,859 --> 00:22:33,960 FOR THEIR 443 00:22:33,960 --> 00:22:34,528 INCREDIBLE WORK RELATED TO 444 00:22:34,528 --> 00:22:36,263 SICKLE CELL DISEASE. 445 00:22:36,263 --> 00:22:39,132 AGAIN, THAT FUNDAMENTAL 446 00:22:39,132 --> 00:22:41,735 DISCOVERY SCIENCE, INDEED ONE OF 447 00:22:41,735 --> 00:22:45,539 THE PROTOCOLS THAT IS BEING 448 00:22:45,539 --> 00:22:46,940 FUNDED THROUGH CURE SICKLE CELL 449 00:22:46,940 --> 00:22:49,910 TO ACTUALLY TAKE ARLEN TEE VIRUS 450 00:22:49,910 --> 00:22:53,547 SHORT HAIRPIN TO KNOCK DOWN 451 00:22:53,547 --> 00:22:55,715 BC11A AS A MEANS OF POTENTIALLY 452 00:22:55,715 --> 00:22:59,219 TREATING SICKLE CELL DISEASE. 453 00:22:59,219 --> 00:23:02,556 AND IT'S COME THROUGH THAT WHOLE 454 00:23:02,556 --> 00:23:03,823 VIRTUOUS CYCLE. 455 00:23:03,823 --> 00:23:05,792 WANT TO ACKNOWLEDGE THE 456 00:23:05,792 --> 00:23:07,761 EXTRAMURAL TEAMS THAT HAVE 457 00:23:07,761 --> 00:23:11,698 REALLY DRIVEN THAT WHOLE 458 00:23:11,698 --> 00:23:17,704 PROCESS, TRACI MONDORO, JULIE 459 00:23:17,704 --> 00:23:18,572 PANEPINTO, COMPLEMENTING THE 460 00:23:18,572 --> 00:23:21,207 WORK OF JOHN TISDALE, AGAIN 461 00:23:21,207 --> 00:23:23,176 YOU'LL HEAR FROM COURTNEY IN HER 462 00:23:23,176 --> 00:23:27,080 TALK. 463 00:23:27,080 --> 00:23:28,648 I THINK IT'S A NICE BLENDING OF 464 00:23:28,648 --> 00:23:30,417 THE EXTRAMURAL AND EXTRAMURAL TO 465 00:23:30,417 --> 00:23:33,219 DO SOMETHING TRANSFORMATIONAL 466 00:23:33,219 --> 00:23:35,388 THAT WE HOPE TO CONTINUE TO 467 00:23:35,388 --> 00:23:42,028 EXPAND UPON. 468 00:23:42,028 --> 00:23:43,096 ANOTHER SORT OF UPDATE AND 469 00:23:43,096 --> 00:23:44,097 OPPORTUNITY RELATES TO WOMEN'S 470 00:23:44,097 --> 00:23:47,067 HEALTH. 471 00:23:47,067 --> 00:23:52,405 AND THAT PICTURE THERE IS A 472 00:23:52,405 --> 00:23:54,240 CEREMONY THAT WAS HELD WHEN 473 00:23:54,240 --> 00:23:56,443 PRESIDENT BIDEN SIGNED AN 474 00:23:56,443 --> 00:23:58,311 EXECUTIVE ORDER RELATED TO 475 00:23:58,311 --> 00:23:59,746 WOMEN'S HEALTH RESEARCH 476 00:23:59,746 --> 00:24:04,217 INITIATIVE AT THE WHITE HOUSE. 477 00:24:04,217 --> 00:24:05,752 WE HAD THE PRIVILEGE OF BEING 478 00:24:05,752 --> 00:24:08,688 INVITE TO THAT CEREMONY, WHERE 479 00:24:08,688 --> 00:24:11,458 HE OUTLINED A BOLD VISION FOR 480 00:24:11,458 --> 00:24:12,192 WOMEN'S HEALTH. 481 00:24:12,192 --> 00:24:14,728 ACTUALLY TALKING ABOUT THE 482 00:24:14,728 --> 00:24:17,897 BILLION DOLLARS A YEAR FOR ABOUT 483 00:24:17,897 --> 00:24:19,532 10 YEARS ABOVE WHAT WE ALREADY 484 00:24:19,532 --> 00:24:20,900 ARE INVESTING. 485 00:24:20,900 --> 00:24:22,469 WHETHER THAT WILL TRANSLATE INTO 486 00:24:22,469 --> 00:24:24,170 ACTUAL APPROPRIATION STILL 487 00:24:24,170 --> 00:24:28,074 REMAINS TO BE SEEN, BUT AGAIN 488 00:24:28,074 --> 00:24:29,275 NEVERTHELESS A BOLD AND 489 00:24:29,275 --> 00:24:32,779 IMPORTANT VISION. 490 00:24:32,779 --> 00:24:35,715 AND TOWARD THAT END, THIS IS 491 00:24:35,715 --> 00:24:37,350 CERTAINLY SOMETHING THAT 492 00:24:37,350 --> 00:24:38,518 REFLECTS THE SCIENTIFIC 493 00:24:38,518 --> 00:24:40,420 OPPORTUNITY RELEVANT TO THE 494 00:24:40,420 --> 00:24:41,287 NHLBI. 495 00:24:41,287 --> 00:24:44,557 ONE OF THE THINGS THAT I THINK 496 00:24:44,557 --> 00:24:49,062 WE'RE ALL AWARE OF AS CLINICIANS 497 00:24:49,062 --> 00:24:51,331 IS THE SEX-BASED DIFFERENCES IN 498 00:24:51,331 --> 00:24:54,300 MALE AND FEMALES IN TERMS OF 499 00:24:54,300 --> 00:24:55,969 THAT CARDIOVASCULAR RISK 500 00:24:55,969 --> 00:24:59,139 PROFILE, AND HOW THERE'S BEEN 501 00:24:59,139 --> 00:25:02,509 ALL THIS WORKING HYPOTHESIS 502 00:25:02,509 --> 00:25:04,044 SINCE PROBABLY I WAS A MEDICAL 503 00:25:04,044 --> 00:25:07,647 STUDENT THAT WOMEN ARE PROTECTED 504 00:25:07,647 --> 00:25:10,383 IN SOME WAY FROM THAT RISK UNTIL 505 00:25:10,383 --> 00:25:13,787 MID LIFE AND MENOPAUSE AND 506 00:25:13,787 --> 00:25:16,523 PRESUMABLY THE LOSS OF SOME OF 507 00:25:16,523 --> 00:25:18,058 THOSE -- THAT PROTECTIVE EFFECTS 508 00:25:18,058 --> 00:25:19,793 OF ESTROGEN, AT LEAST THAT WAS 509 00:25:19,793 --> 00:25:26,366 THE HYPOTHESIS, SUCH THAT THAT 510 00:25:26,366 --> 00:25:27,467 PROTECTIVE EFFECT IS LOST. 511 00:25:27,467 --> 00:25:29,636 BUT I NEVER WAS CONVINCED WE 512 00:25:29,636 --> 00:25:31,504 REALLY HAD A GOOD MECHANISTIC 513 00:25:31,504 --> 00:25:32,272 UNDERSTANDING OF WHY THAT 514 00:25:32,272 --> 00:25:37,077 ACTUALLY IS. 515 00:25:37,077 --> 00:25:40,547 INDEED, IN SOME WAYS, THE FIRST 516 00:25:40,547 --> 00:25:42,549 WOMEN'S HEALTH INITIATIVE NOW, 517 00:25:42,549 --> 00:25:44,484 WHAT, ALMOST 30 YEARS AGO, 518 00:25:44,484 --> 00:25:47,921 LOOKED AT THE INFLUENCE OF 519 00:25:47,921 --> 00:25:51,624 PROVIDING HORMONE REPLACEMENT 520 00:25:51,624 --> 00:25:53,226 THERAPY, PRESUMABLY ON THAT 521 00:25:53,226 --> 00:25:53,593 HYPOTHESIS. 522 00:25:53,593 --> 00:25:54,694 WELL, IF WE GIVE BACK THE 523 00:25:54,694 --> 00:25:57,197 ESTROGEN AND PROGESTERONE IN A 524 00:25:57,197 --> 00:25:57,964 COMBINATION, PERHAPS THAT WOULD 525 00:25:57,964 --> 00:25:59,833 BE A GOOD THING. 526 00:25:59,833 --> 00:26:01,468 AS EVERYONE IN THIS AUDIENCE 527 00:26:01,468 --> 00:26:02,769 KNOWS, IT DIDN'T QUITE WORK OUT 528 00:26:02,769 --> 00:26:05,305 LIKE THAT HYPOTHESIS THOUGHT. 529 00:26:05,305 --> 00:26:06,740 INDEED, THERE APPEARED TO BE A 530 00:26:06,740 --> 00:26:10,009 LOT OF HARM POTENTIALLY TO THE 531 00:26:10,009 --> 00:26:11,277 HORMONE REPLACEMENT THERAPY 532 00:26:11,277 --> 00:26:13,379 REGIMES THAT WERE USED IN THE 533 00:26:13,379 --> 00:26:15,148 90s. 534 00:26:15,148 --> 00:26:17,450 AND SO I THINK THAT SENT US BACK 535 00:26:17,450 --> 00:26:19,185 TO THE DRAWS BOARD ON SOME OF 536 00:26:19,185 --> 00:26:28,895 THOSE HYPOTHESES. 537 00:26:28,895 --> 00:26:31,731 AND RELATED TO THAT, 538 00:26:31,731 --> 00:26:36,102 EPIDEMIOLOGIC STUDIES HAVE ALSO 539 00:26:36,102 --> 00:26:37,537 STARTED TO DIG A LITTLE FURTHER 540 00:26:37,537 --> 00:26:38,505 INTO WHAT THAT MEANS TO HAVE 541 00:26:38,505 --> 00:26:41,374 THAT MID LIFE TRANSITION INTO 542 00:26:41,374 --> 00:26:41,975 MENOPAUSE. 543 00:26:41,975 --> 00:26:45,845 I ALWAYS FEE K FEEL KIND OF AWKS 544 00:26:45,845 --> 00:26:47,447 AN OLD MALE GUY TALKING ABOUT 545 00:26:47,447 --> 00:26:48,148 MENOPAUSE, CERTAINLY NOT 546 00:26:48,148 --> 00:26:52,418 SOMETHING THAT I'M EXPERT IN, 547 00:26:52,418 --> 00:26:55,021 BUT CLEARLY, THERE'S A 548 00:26:55,021 --> 00:26:57,123 RELATIONSHIP BETWEEN A LOT OF 549 00:26:57,123 --> 00:26:59,526 THOSE SYMPTOMS THAT RELATE TO 550 00:26:59,526 --> 00:27:02,028 THAT TRANSITION AND RISK. 551 00:27:02,028 --> 00:27:06,733 AND SO THAT WAS ALREADY A CLUE 552 00:27:06,733 --> 00:27:09,135 THAT THAT TRANSITION IS 553 00:27:09,135 --> 00:27:12,105 INFLUENCING THESE PROCESSES BUT 554 00:27:12,105 --> 00:27:12,972 MECHANISTICALLY STILL REMAIN A 555 00:27:12,972 --> 00:27:17,010 BIT UNCLEAR. 556 00:27:17,010 --> 00:27:18,545 AND TOWARD THAT END AS PART OF 557 00:27:18,545 --> 00:27:19,879 THIS VIRTUOUS CYCLE, I ALWAYS 558 00:27:19,879 --> 00:27:22,348 FOUND IT INTRIGUING THAT IF THAT 559 00:27:22,348 --> 00:27:25,118 FIRST CLINICAL TRIAL WHERE WE 560 00:27:25,118 --> 00:27:27,620 JUST TOOK A SIMPLE HYPOTHESIS 561 00:27:27,620 --> 00:27:29,055 DIDN'T KIND OF WORK OUT, WE NEED 562 00:27:29,055 --> 00:27:30,590 TO GO BACK TO THE BENCH AND 563 00:27:30,590 --> 00:27:34,394 REALLY START TO DIG DEEPER AND 564 00:27:34,394 --> 00:27:35,395 UNDERSTAND WHAT'S GOING ON IN 565 00:27:35,395 --> 00:27:36,763 TERMS OF THE MOLECULAR PATHWAYS 566 00:27:36,763 --> 00:27:38,131 THAT MAY BE DRIVING SOME OF 567 00:27:38,131 --> 00:27:41,534 THESE THINGS. 568 00:27:41,534 --> 00:27:42,735 AND CLEARLY ALL THAT 569 00:27:42,735 --> 00:27:45,672 SYMPTOMATOLOGY, THE HOT FLASHES, 570 00:27:45,672 --> 00:27:47,640 THE NIGHT SWEATS, ALL THESE 571 00:27:47,640 --> 00:27:49,943 THINGS WERE SUGGESTIVE OF A 572 00:27:49,943 --> 00:27:52,445 PERTURBATION OF THERMAL 573 00:27:52,445 --> 00:27:53,880 REGULATORY CONTROL THAT THEN 574 00:27:53,880 --> 00:27:57,917 SPEAKS TO BLOOD VESSELS AS 575 00:27:57,917 --> 00:27:59,118 THERMAL REGULATION HAS EVOLVED 576 00:27:59,118 --> 00:27:59,786 TO DO. 577 00:27:59,786 --> 00:28:02,722 AND HOW THAT'S PERTURBED IN THE 578 00:28:02,722 --> 00:28:05,158 CONTEXT OF HORMONAL TRANSITIONS. 579 00:28:05,158 --> 00:28:07,126 THERE ARE CERTAIN MOLECULES THAT 580 00:28:07,126 --> 00:28:08,761 MEDIATE SOME OF THOSE, AND SOME 581 00:28:08,761 --> 00:28:11,798 OF THOSE HAVE BEEN OBSERVED NOW 582 00:28:11,798 --> 00:28:16,536 WITH NEUROKININ 3, AND 583 00:28:16,536 --> 00:28:18,004 DEMONSTRATING THE LINKAGES OF 584 00:28:18,004 --> 00:28:21,774 THE WHOLE HYPOTHAT LA MACK BY 585 00:28:21,774 --> 00:28:26,346 TEU TERRY GONADAL AXIS THAT 586 00:28:26,346 --> 00:28:28,948 IDENTIFIES THOSE PATHWAYS, A KEY 587 00:28:28,948 --> 00:28:29,983 MEDIATOR OF SOME OF THOSE 588 00:28:29,983 --> 00:28:32,819 FACTORS. 589 00:28:32,819 --> 00:28:34,153 WITH THAT BASIC SCIENCE 590 00:28:34,153 --> 00:28:36,723 UNDERSTANDING AND TARGETING OF 591 00:28:36,723 --> 00:28:42,996 THAT AS A KEY MEDIATOR, 592 00:28:42,996 --> 00:28:44,864 FDA-APPROVED THERAPIES FOR 593 00:28:44,864 --> 00:28:46,633 NON-HORMONAL AGENTS THAT ARE 594 00:28:46,633 --> 00:28:49,402 NOVEL THAT MAY ACTUALLY MODULATE 595 00:28:49,402 --> 00:28:55,208 THOSE VASOMOTOR SYMPTOMS. 596 00:28:55,208 --> 00:28:56,709 SO IT'S NOT CLEAR TO ME WHETHER 597 00:28:56,709 --> 00:28:58,811 THAT HAS IMPLICATIONS BEYOND THE 598 00:28:58,811 --> 00:28:59,879 VASOMOTOR SYMPTOMS AND AFFECTS 599 00:28:59,879 --> 00:29:02,081 OTHER FORMS OF THE DISEASE, BUT 600 00:29:02,081 --> 00:29:04,717 AT LEAST THERE ARE NOW SAFE AND 601 00:29:04,717 --> 00:29:07,553 EFFECTIVE THERAPIES THAT ARE 602 00:29:07,553 --> 00:29:10,290 EMERGING AS WELL AS MORE 603 00:29:10,290 --> 00:29:12,458 APPROPRIATE WAYS OF DELIVERING 604 00:29:12,458 --> 00:29:14,294 ESTROGEN THAT WE HOPE ARE 605 00:29:14,294 --> 00:29:23,636 ADDRESSING THE NEEDS OF WOMEN. 606 00:29:23,636 --> 00:29:24,904 SO THOSE THEMES OF ADDRESSING 607 00:29:24,904 --> 00:29:25,905 WOMEN'S HEALTH ACROSS THE 608 00:29:25,905 --> 00:29:27,240 LIFESPAN THAT ARE OUTLINED IN 609 00:29:27,240 --> 00:29:30,076 THAT VISION IN THE EXECUTIVE 610 00:29:30,076 --> 00:29:32,378 ORDER, I THINK ARE QUITE 611 00:29:32,378 --> 00:29:36,849 RELEVANT TO OUR PORTFOLIO. 612 00:29:36,849 --> 00:29:40,887 I BELIEVE WE CAN START TO PROBE 613 00:29:40,887 --> 00:29:43,089 EVEN FURTHER ABOUT WHAT THOSE 614 00:29:43,089 --> 00:29:45,591 TRANSITION POINTS ARE, AND 615 00:29:45,591 --> 00:29:46,926 AGAIN, HOW WE CAN BEST ADDRESS 616 00:29:46,926 --> 00:29:50,296 THE NEEDS OF WOMEN IN MID LIFE. 617 00:29:50,296 --> 00:29:53,466 AND TOWARD THAT END, IT'S CLEAR 618 00:29:53,466 --> 00:29:56,869 THAT THERE IS THIS RELATIONSHIP 619 00:29:56,869 --> 00:29:58,938 BETWEEN PARTICULARLY EARLY 620 00:29:58,938 --> 00:30:02,108 MENOPAUSE AND RISK THAT AGAIN 621 00:30:02,108 --> 00:30:05,078 SUGGESTS ITS CRITICAL ROLE. 622 00:30:05,078 --> 00:30:07,814 AND INDEED, THAT SEEMS TO 623 00:30:07,814 --> 00:30:10,883 CORRESPOND TO CHANGES IN 624 00:30:10,883 --> 00:30:12,618 VASCULAR FUNCTION AS A HARBINGER 625 00:30:12,618 --> 00:30:14,153 THAT THERE IS SOMETHING GOING ON 626 00:30:14,153 --> 00:30:15,989 WITH VASCULAR DISEASE DURING 627 00:30:15,989 --> 00:30:16,556 THAT TRANSITION. 628 00:30:16,556 --> 00:30:19,292 AND INDEED MAY EVEN INTERSECT 629 00:30:19,292 --> 00:30:21,027 WITH SOME OF THE RACIAL 630 00:30:21,027 --> 00:30:23,563 DISPARITIES WE SEE AS WELL THAT 631 00:30:23,563 --> 00:30:24,864 AMPLIFIES SOME OF THESE 632 00:30:24,864 --> 00:30:32,205 ELEMENTS. 633 00:30:32,205 --> 00:30:34,173 AND INDEED SOME OF THE MOLECULAR 634 00:30:34,173 --> 00:30:37,010 FINGERPRINTS APPEAR TO BE 635 00:30:37,010 --> 00:30:39,846 EMERGING IN WHICH THESE 636 00:30:39,846 --> 00:30:41,481 TRANSITIONS MIGHT AFFECT BOTH 637 00:30:41,481 --> 00:30:44,350 THE EPIGENOME AND THE 638 00:30:44,350 --> 00:30:44,684 TRANSCRIPTOME. 639 00:30:44,684 --> 00:30:49,155 PERHAPS NOT TOO SURPRISING, 640 00:30:49,155 --> 00:30:53,726 GIVEN THE HORMONES -- BASICALLY 641 00:30:53,726 --> 00:30:55,328 LIGANDS CAPABLE OF MODULATING 642 00:30:55,328 --> 00:30:55,828 THE GENOME. 643 00:30:55,828 --> 00:30:59,966 AND TOWARD THAT END, AGAIN, 644 00:30:59,966 --> 00:31:02,035 THESE FACTORS ARE STARTING TO 645 00:31:02,035 --> 00:31:03,269 EMERGE AS MOLECULAR PATHWAYS 646 00:31:03,269 --> 00:31:11,044 DURING THIS TRANSITION. 647 00:31:11,044 --> 00:31:13,646 INDEED, SOME SENSE THAT THIS 648 00:31:13,646 --> 00:31:17,483 COULD INTERFACE WITH ACCELERATED 649 00:31:17,483 --> 00:31:21,320 AND BIOLOGICAL AGING RELATED TO 650 00:31:21,320 --> 00:31:25,091 THESE TRANSITIONS, INDEED EVEN 651 00:31:25,091 --> 00:31:26,459 INCLUDING TELEMERE LENGTH. 652 00:31:26,459 --> 00:31:30,730 SO CLEARLY THE BIOLOGY, SYSTEMS 653 00:31:30,730 --> 00:31:31,931 BIOLOGY IS BEING MODULATED IN 654 00:31:31,931 --> 00:31:34,867 THE CONTEXT OF THIS TRANSITION. 655 00:31:34,867 --> 00:31:39,772 AND I THINK W WE'RE JUST TIPPING 656 00:31:39,772 --> 00:31:42,308 THE ICEBERG IN REALLY 657 00:31:42,308 --> 00:31:44,710 UNDERSTANDING THE MAGNITUDE OF 658 00:31:44,710 --> 00:31:48,748 THESE CHANGES ON SYSTEMS. 659 00:31:48,748 --> 00:31:49,315 THAT 660 00:31:49,315 --> 00:31:52,919 AFFECT WOMEN AND WE HAD THE 661 00:31:52,919 --> 00:31:54,787 CAPABILITY NOW TO CHARACTERIZE 662 00:31:54,787 --> 00:31:57,390 THAT IN A MORE PROFOUND AND 663 00:31:57,390 --> 00:31:59,592 DEEPER WAY. 664 00:31:59,592 --> 00:32:01,994 AND AGAIN, THERE ARE HINTS THERE 665 00:32:01,994 --> 00:32:05,631 AS WELL, AS WE THINK ABOUT THIS 666 00:32:05,631 --> 00:32:07,166 POTENTIALLY FROM A MULTI-OMIC 667 00:32:07,166 --> 00:32:11,737 AND SYSTEMS BIOLOGY APPROACH TO 668 00:32:11,737 --> 00:32:13,606 UNDERSTANDING THAT TRANSITION 669 00:32:13,606 --> 00:32:18,311 ACROSS THE LIFESPAN THAT COULD 670 00:32:18,311 --> 00:32:19,312 HAVE IMPLICATIONS AND IS 671 00:32:19,312 --> 00:32:20,947 STARTING TO REVEAL CERTAIN 672 00:32:20,947 --> 00:32:22,348 MOLECULAR PATHWAYS THAT MAY BE 673 00:32:22,348 --> 00:32:25,952 ALTERED IN THAT CONTEXT. 674 00:32:25,952 --> 00:32:30,556 AGAIN, GIVEN SEX-SPECIFIC 675 00:32:30,556 --> 00:32:32,325 DIFFERENCES THAT ARE PART OF 676 00:32:32,325 --> 00:32:34,494 THAT TRAJECTORY THAT WOMEN HAVE 677 00:32:34,494 --> 00:32:35,795 THAT ARE CLEARLY DISTINCT FROM 678 00:32:35,795 --> 00:32:36,562 MEN. 679 00:32:36,562 --> 00:32:39,098 AND THAT MAY HAVE LONG TERM 680 00:32:39,098 --> 00:32:41,267 IMPLICATIONS. 681 00:32:41,267 --> 00:32:44,470 AND SO THAT WOMEN'S HEALTH 682 00:32:44,470 --> 00:32:47,406 INITIATIVE, WE THINK COULD BE A 683 00:32:47,406 --> 00:32:49,909 GREAT OPPORTUNITY TO START TO 684 00:32:49,909 --> 00:32:52,745 THINK ABOUT HUMAN SYSTEMS 685 00:32:52,745 --> 00:32:54,847 BIOLOGY IN A VERY DEEP WAY AT 686 00:32:54,847 --> 00:32:59,118 SCALE IN WAYS THAT WE THINK 687 00:32:59,118 --> 00:33:01,053 COULD CONTRIBUTE TO ENHANCING 688 00:33:01,053 --> 00:33:01,420 WOMEN'S HEALTH. 689 00:33:01,420 --> 00:33:04,457 LET ME NOW PIVOT FROM WOMEN'S 690 00:33:04,457 --> 00:33:08,828 HEALTH TO ANOTHER AREA THAT WE 691 00:33:08,828 --> 00:33:11,464 THINK IS A GROWING OPPORTUNITY. 692 00:33:11,464 --> 00:33:14,767 WE ALLUDED TO THE ADVANCES OF 693 00:33:14,767 --> 00:33:19,205 THE LUNG DIVISION UNDER 694 00:33:19,205 --> 00:33:21,874 DR. KILEY'S LEADERSHIP, AND ONE 695 00:33:21,874 --> 00:33:24,911 OF THE AREAS THAT WE'VE BEEN 696 00:33:24,911 --> 00:33:27,980 DIALOGUING ABOUT AS THE LUNG 697 00:33:27,980 --> 00:33:29,415 DIVISION DOES ITS STRATEGIC 698 00:33:29,415 --> 00:33:33,219 VISION REFRESH IS REALLY DRIVING 699 00:33:33,219 --> 00:33:35,087 GREATER DEPTH IN THE MOLECULAR 700 00:33:35,087 --> 00:33:38,157 BASIS OF CHRONIC LUNG DISEASE, 701 00:33:38,157 --> 00:33:39,992 AND MOVING THROUGH THAT SAME 702 00:33:39,992 --> 00:33:46,232 VIRTUAL CYCLE. 703 00:33:46,232 --> 00:33:47,333 AND TOWARD THAT END, ONE OF THE 704 00:33:47,333 --> 00:33:48,968 AREAS THAT WE'VE BEEN TRYING TO 705 00:33:48,968 --> 00:33:54,874 HIGH LIGLIGHT RELATES TO IDIOPAC 706 00:33:54,874 --> 00:33:55,541 PULMONARY FIBROSIS. 707 00:33:55,541 --> 00:33:56,742 SOME OF YOU HAVE HEARD ME TALK 708 00:33:56,742 --> 00:33:58,044 ABOUT PREVIOUSLY WHERE IT'S ONE 709 00:33:58,044 --> 00:34:01,881 OF THOSE SORT OF VEXING 710 00:34:01,881 --> 00:34:04,517 CHALLENGES THAT WE'VE HAD FOR 711 00:34:04,517 --> 00:34:05,685 SOME TIME WHERE THESE 712 00:34:05,685 --> 00:34:08,988 INDIVIDUALS, BY THE TIME THEY'RE 713 00:34:08,988 --> 00:34:11,224 DIAGNOSED, ON OXYGEN, HAVE THE 714 00:34:11,224 --> 00:34:15,194 HORRIBLE PROGNOSIS, AND THAT 715 00:34:15,194 --> 00:34:16,229 PROGNOSIS HASN'T CHANGED MUCH 716 00:34:16,229 --> 00:34:17,964 OVER TIME, AND SO THIS IS REALLY 717 00:34:17,964 --> 00:34:22,101 AN AREA THAT'S INCREDIBLE AND I 718 00:34:22,101 --> 00:34:24,203 THINK WE HAVE TO REDOUBLE OUR 719 00:34:24,203 --> 00:34:29,342 EFFORTS. 720 00:34:29,342 --> 00:34:34,380 TOWARD THAT END, WE'RE INTRIGUED 721 00:34:34,380 --> 00:34:36,215 BY, AGAIN, EMERGING BASIC 722 00:34:36,215 --> 00:34:38,618 SCIENCE, AND SOME OF THE THINGS 723 00:34:38,618 --> 00:34:42,121 THAT COUNCIL HAS ALREADY 724 00:34:42,121 --> 00:34:43,356 REVIEWED A BIT IN TERMS OF THE 725 00:34:43,356 --> 00:34:44,857 LUNG MAP AND APPRECIATING HOW 726 00:34:44,857 --> 00:34:45,858 MANY DIFFERENT KINDS OF CELLS 727 00:34:45,858 --> 00:34:47,827 ARE IN THE LUNG. 728 00:34:47,827 --> 00:34:51,964 AND INCLUDING PROGENITOR CELLS 729 00:34:51,964 --> 00:34:55,401 AND A GREAT REPARATIVE CAPACITY, 730 00:34:55,401 --> 00:34:57,870 INTERACTION WITH THE IMMUNE 731 00:34:57,870 --> 00:34:59,205 SYSTEM, AND ONE OF THE THINGS 732 00:34:59,205 --> 00:35:02,275 THAT WAS VERY INTRIGUING WAS 733 00:35:02,275 --> 00:35:06,212 THIS EXPERIMENT IN WHICH IT 734 00:35:06,212 --> 00:35:11,784 TARGETED SELECTIVELY CELL NE 735 00:35:11,784 --> 00:35:13,552 SENESCENCE, AND THIS WAS A MODEL 736 00:35:13,552 --> 00:35:18,991 THAT RESULTED IN ENHANCED LUNG 737 00:35:18,991 --> 00:35:20,426 FIBROSIS. 738 00:35:20,426 --> 00:35:21,827 AGAIN, INTRIGUING AS TO HOW WE 739 00:35:21,827 --> 00:35:28,067 GET TO THAT END STAGE OF IPF AND 740 00:35:28,067 --> 00:35:30,036 HOW THAT MAY RELATE TO THE 741 00:35:30,036 --> 00:35:31,671 ABILITY OF THE LUNG, WHICH IS 742 00:35:31,671 --> 00:35:33,339 REALLY AT THE FRONT LINE OF 743 00:35:33,339 --> 00:35:35,541 EXPOSURES OF A LOT OF THINGS 744 00:35:35,541 --> 00:35:37,476 THAT ARE INJURIOUS IN ITS 745 00:35:37,476 --> 00:35:40,112 CAPACITY TO REPAIR ITSELF. 746 00:35:40,112 --> 00:35:40,313 OVER 747 00:35:40,313 --> 00:35:41,514 TIME. 748 00:35:41,514 --> 00:35:43,616 AND AS WE TRY TO UNDERSTAND 749 00:35:43,616 --> 00:35:46,452 THESE PROCESSES AND 750 00:35:46,452 --> 00:35:47,887 SUSCEPTIBILITIES AND TRY TO 751 00:35:47,887 --> 00:35:48,854 UNDERSTAND THINGS AT THE 752 00:35:48,854 --> 00:35:53,793 EARLIEST ONSET, THE FAMILIAL 753 00:35:53,793 --> 00:35:57,263 MODELS SEEMS TO HOLD A LOT OF 754 00:35:57,263 --> 00:35:58,798 PROMISE, GIVEN THOSE 755 00:35:58,798 --> 00:35:59,532 PREDISPOSITIONS. 756 00:35:59,532 --> 00:36:04,670 AND IT'S INTRIGUING THAT MUCH OF 757 00:36:04,670 --> 00:36:07,673 THE EARLY WORK RELATES TO 758 00:36:07,673 --> 00:36:08,941 POTENTIAL VARIANTS IN GENES THAT 759 00:36:08,941 --> 00:36:15,314 ARE INVOLVED IN SENESCENCE AND 760 00:36:15,314 --> 00:36:17,416 TELOMERE LENGTH LIKE TERT, IN 761 00:36:17,416 --> 00:36:19,485 WHICH THESE VARIANTS ARE 762 00:36:19,485 --> 00:36:21,887 ASSOCIATED WITH RISK IN THESE 763 00:36:21,887 --> 00:36:26,158 PATIENT POPULATIONS, AGAIN 764 00:36:26,158 --> 00:36:28,027 CONSISTENT PERHAPS WITH SOME 765 00:36:28,027 --> 00:36:34,567 ACCELERATED AGING. 766 00:36:34,567 --> 00:36:37,303 SO REALLY WE DO HAVE THAT 767 00:36:37,303 --> 00:36:37,803 OPPORTUNITY POTENTIALLY 768 00:36:37,803 --> 00:36:39,605 SUGGESTED BY THOSE FAMILIAL 769 00:36:39,605 --> 00:36:41,040 CASES, PERHAPS FIND FOLKS WHO 770 00:36:41,040 --> 00:36:43,342 ARE SUSCEPTIBLE AND GETTING AN 771 00:36:43,342 --> 00:36:44,643 EARLIER ONSET OF THE DISEASE TO 772 00:36:44,643 --> 00:36:46,278 GET UPSTREAM TO REALLY START TO 773 00:36:46,278 --> 00:36:49,882 SEE WHAT MIGHT BE PRE-CLINICAL 774 00:36:49,882 --> 00:36:51,417 AND UNDERSTAND THOSE MEDIATOR 775 00:36:51,417 --> 00:36:53,052 PATHWAYS IN A WAY THAT WE MIGHT 776 00:36:53,052 --> 00:36:55,488 BE ABLE TO INTERRUPT OR PREEMPT 777 00:36:55,488 --> 00:37:03,229 OR MODIFY DISEASE COURSE. 778 00:37:03,229 --> 00:37:05,965 IN THIS REGARD, INTRIGUED BY 779 00:37:05,965 --> 00:37:06,599 EMERGING OPPORTUNITIES WHERE 780 00:37:06,599 --> 00:37:08,267 EVERYTHING HAS GOT TO BE AN OMIC 781 00:37:08,267 --> 00:37:09,902 THESE DAYS, SO THE RADIOLOGIST 782 00:37:09,902 --> 00:37:13,305 DIDN'T WANT TO BE LEFT OUT SO 783 00:37:13,305 --> 00:37:17,777 NOW THERE'S RADIOMICS, MACHINE 784 00:37:17,777 --> 00:37:18,544 LEARNING, ARTIFICIAL 785 00:37:18,544 --> 00:37:19,879 INTELLIGENCE, TO BE ABLE TO 786 00:37:19,879 --> 00:37:24,984 DISCERN FEATURES FROM VARIOUS 787 00:37:24,984 --> 00:37:35,294 TESTS RAID YO LI RADIOLOGIC TES, 788 00:37:35,294 --> 00:37:38,030 WHETHER THAT CAN HELP US GET 789 00:37:38,030 --> 00:37:38,931 UPSTREAM TO IT. 790 00:37:38,931 --> 00:37:40,766 IT WOULD SEEM THAT THIS IS A 791 00:37:40,766 --> 00:37:41,534 SUBSTANTIAL OPPORTUNITY AS WE 792 00:37:41,534 --> 00:37:43,068 LOOK TO THE STRATEGIC VISION 793 00:37:43,068 --> 00:37:45,237 OVER THE NEXT FIVE TO 10 YEARS, 794 00:37:45,237 --> 00:37:49,074 THE APPLICATION OF AI TO IMAGING 795 00:37:49,074 --> 00:37:53,312 AND ITS IMPLICATIONS FOR 796 00:37:53,312 --> 00:37:54,346 CLINICAL MEDICINE, WE THINK ARE 797 00:37:54,346 --> 00:37:56,749 GOING TO BE PROFOUND, AND I 798 00:37:56,749 --> 00:37:58,584 BELIEVE THAT NHLBI NEEDS TO MAKE 799 00:37:58,584 --> 00:38:01,320 INVESTMENTS IN THAT SPACE, NIH 800 00:38:01,320 --> 00:38:02,621 NEEDS TO MAKE INVESTMENTS IN 801 00:38:02,621 --> 00:38:06,592 THAT SPACE APPLICABLE TO OUR 802 00:38:06,592 --> 00:38:11,931 PORTFOLIO. 803 00:38:11,931 --> 00:38:15,100 INDEED SOME INVESTIGATORS AGAIN 804 00:38:15,100 --> 00:38:16,335 RELATED -- FUNDED BY NHLBI, 805 00:38:16,335 --> 00:38:18,704 THEY'RE STARTING TO EXPLORE THE 806 00:38:18,704 --> 00:38:20,139 SPACE AND TRYING TO IDENTIFY 807 00:38:20,139 --> 00:38:27,813 SOME OF THESE EARLY CHANGES, 808 00:38:27,813 --> 00:38:30,416 RADIOGRAPHICALLY, INTERSTITIAL 809 00:38:30,416 --> 00:38:31,851 LUNG ABNORMALITIES, TRYING TO 810 00:38:31,851 --> 00:38:33,152 CHARACTERIZE IT IN WAYS THAT WE 811 00:38:33,152 --> 00:38:36,222 HOPE ARE PRIOR TO A FINAL 812 00:38:36,222 --> 00:38:39,525 DIAGNOSIS OF PULMONARY FIBROSIS, 813 00:38:39,525 --> 00:38:40,926 WOULD GIVE US MORE OF A SENSE OF 814 00:38:40,926 --> 00:38:42,294 THOSE EARLY CHANGES IN WHICH WE 815 00:38:42,294 --> 00:38:44,330 MIGHT BE ABLE TO INTERVENE AND 816 00:38:44,330 --> 00:38:45,397 MAKE A DIFFERENCE. 817 00:38:45,397 --> 00:38:49,134 AND AGAIN, SOME OF THOSE SAME 818 00:38:49,134 --> 00:38:53,405 AREAS OF CONVERGENCE, OLDER AGE, 819 00:38:53,405 --> 00:38:54,807 EXPOSURES, THINGS LIKE SMOKING, 820 00:38:54,807 --> 00:38:59,945 SHORTER TELOMERE LENGTHS, INDEED 821 00:38:59,945 --> 00:39:03,215 SOME OF THE CANDIDATES GENES 822 00:39:03,215 --> 00:39:06,185 APPEAR TO BE PLAYING A ROLE, ALL 823 00:39:06,185 --> 00:39:07,520 SUGGESTING THAT WE'RE GETTING 824 00:39:07,520 --> 00:39:11,557 CLOSER AND CLOSER TO STARTING TO 825 00:39:11,557 --> 00:39:14,927 GET UPSTREAM AND GET TOWARD 826 00:39:14,927 --> 00:39:17,563 FACTORS THAT MAY BE PREDISPOSING 827 00:39:17,563 --> 00:39:19,865 AND IDENTIFYING THOSE AT RISK AS 828 00:39:19,865 --> 00:39:26,105 WELL AS MEDIATORS. 829 00:39:26,105 --> 00:39:27,273 THIS IS MOVING FORWARD IN WAYS 830 00:39:27,273 --> 00:39:29,275 THAT ARE INTRIGUING IN WHICH 831 00:39:29,275 --> 00:39:32,645 PICKING UP SOME OF THESE IMAGING 832 00:39:32,645 --> 00:39:33,445 BIOMARKERS START TO BE RELATED 833 00:39:33,445 --> 00:39:36,382 TO SOME OF THESE MEDIATING 834 00:39:36,382 --> 00:39:37,583 PATHWAYS, CERTAINLY INFLAMMATION 835 00:39:37,583 --> 00:39:40,953 AND OTHERS. 836 00:39:40,953 --> 00:39:44,123 AND THESE ARE COMING FROM OUR 837 00:39:44,123 --> 00:39:48,627 OBSERVATIONAL COHORTS, ARIC, 838 00:39:48,627 --> 00:39:50,496 FRAMINGHAM, MESA, WHERE WE HAVE 839 00:39:50,496 --> 00:39:53,098 THOSE PROSPECTIVE RADIOLOGICAL 840 00:39:53,098 --> 00:39:56,602 CHARACTERIZATION OF POPULATIONS 841 00:39:56,602 --> 00:39:57,937 RELATED TO THEIR EXPOSURES AND 842 00:39:57,937 --> 00:40:00,439 STARTING TO SEE THESE IN A 843 00:40:00,439 --> 00:40:02,508 PRE-CLINICAL CONTEXT IN THE 844 00:40:02,508 --> 00:40:03,842 COHORTS, AND THAT GIVES US AN 845 00:40:03,842 --> 00:40:06,879 OPPORTUNITY TO THEN APPLY 846 00:40:06,879 --> 00:40:12,451 GENOMICS AND START TO IDENTIFY 847 00:40:12,451 --> 00:40:14,887 GWAS LOCI OR EVEN POLYGENIC RISK 848 00:40:14,887 --> 00:40:16,188 SCORES, AND AGAIN, SOME OF THESE 849 00:40:16,188 --> 00:40:19,024 ARE POPPING UP WITH MEDIATOR 850 00:40:19,024 --> 00:40:20,659 PATHWAYS THAT, AGAIN, MIGHT 851 00:40:20,659 --> 00:40:22,628 RELATE TO REPLICATION AND 852 00:40:22,628 --> 00:40:25,464 SENESCENCE. 853 00:40:25,464 --> 00:40:27,232 AS WELL AS OTHER LOCI. 854 00:40:27,232 --> 00:40:30,536 AND INDEED, THIS IS ANOTHER 855 00:40:30,536 --> 00:40:32,805 COLLECTION OF THE VARIOUS COHORT 856 00:40:32,805 --> 00:40:37,943 STUDIES AND SEGMENTS, INCLUDING 857 00:40:37,943 --> 00:40:45,184 PROGRAMS LIKE COPDGEEP, 858 00:40:45,184 --> 00:40:46,685 SPIROMICS, WHEN WE PUT THAT ALL 859 00:40:46,685 --> 00:40:48,020 TOGETHER IT STARTS TO ENABLE 860 00:40:48,020 --> 00:40:49,421 INVESTIGATORS TO PUT TOGETHER 861 00:40:49,421 --> 00:40:50,656 POLYGENIC RISK SCORES THAT AGAIN 862 00:40:50,656 --> 00:40:52,191 APPEAR TO BE PREDICTIVE IN 863 00:40:52,191 --> 00:40:54,026 HELPING US IDENTIFY WHO'S MOST 864 00:40:54,026 --> 00:40:58,364 AT RISK, WHO WE MIGHT STUDY AND 865 00:40:58,364 --> 00:40:59,131 INTERVENE ON EARLIER IN THE 866 00:40:59,131 --> 00:41:03,435 COURSE OF DISEASE. 867 00:41:03,435 --> 00:41:06,271 AND TOWARD THAT END, 868 00:41:06,271 --> 00:41:09,775 COMPLEMENTING THOSE POPULATION 869 00:41:09,775 --> 00:41:13,812 STUDIES IN THE RADIOMICS WITH IN 870 00:41:13,812 --> 00:41:17,983 DEIN-DEPTH MOLECULAR 871 00:41:17,983 --> 00:41:18,851 CHARACTERIZATION OF TISSUES, 872 00:41:18,851 --> 00:41:21,286 COUNCIL ALSO REVIEWED TODAY A 873 00:41:21,286 --> 00:41:24,556 LUNG MAP AS ONE OF THE SIGNATURE 874 00:41:24,556 --> 00:41:30,029 PROGRAMS OF THE LUNG DIVISION, 875 00:41:30,029 --> 00:41:31,697 STARTING TO GET TO THIS POINT OF 876 00:41:31,697 --> 00:41:33,098 MOLECULAR CHARACTERIZATION OF 877 00:41:33,098 --> 00:41:37,870 TISSUES IN DEPTH IN WAYS THAT 878 00:41:37,870 --> 00:41:39,471 AGAIN ARE STARTING TO IDENTIFY 879 00:41:39,471 --> 00:41:40,706 MEDIATOR PATHWAYS AS SHOWN ON 880 00:41:40,706 --> 00:41:44,943 THE RIGHT, YOU SEE CYTOKINES AND 881 00:41:44,943 --> 00:41:46,478 PROTEINASES AND COLLAGEN, 882 00:41:46,478 --> 00:41:48,781 GIVING, AGAIN, A SENSE OF A 883 00:41:48,781 --> 00:41:51,316 PATHOBIOLOGY. 884 00:41:51,316 --> 00:41:53,852 THAT COULD BE AMENABLE TO 885 00:41:53,852 --> 00:41:56,355 TARGETING, AND SO HOPEFULLY, 886 00:41:56,355 --> 00:41:59,091 OVER THIS NEXT FIVE YEARS, WE 887 00:41:59,091 --> 00:42:02,928 CAN START TO REALLY GET DEEPER 888 00:42:02,928 --> 00:42:06,532 AND GET EARLIER IN UNDERSTANDING 889 00:42:06,532 --> 00:42:08,967 THE PATHOGENESIS OF THIS DISEASE 890 00:42:08,967 --> 00:42:12,337 IN WAYS THAT WE ARE STARTING TO 891 00:42:12,337 --> 00:42:15,841 BE ABLE TO CHARACTERIZE AT 892 00:42:15,841 --> 00:42:18,077 SINGLE CELL RESOLUTION HOPEFULLY 893 00:42:18,077 --> 00:42:20,446 SOME OF THESE MEDIATORS IN THESE 894 00:42:20,446 --> 00:42:22,848 PATHWAYS THAT I HOPE WILL BE 895 00:42:22,848 --> 00:42:25,584 TARGETS FOR DISEASE MODIFYING 896 00:42:25,584 --> 00:42:28,320 THERAPIES OF A NEW GENERATION. 897 00:42:28,320 --> 00:42:31,290 IN A WAY THAT WE HOPE CAN 898 00:42:31,290 --> 00:42:33,258 TRANSFORM THE LIVES OF THOSE 899 00:42:33,258 --> 00:42:35,127 PATIENTS AS WELL. 900 00:42:35,127 --> 00:42:35,561 WITH THESE 901 00:42:35,561 --> 00:42:42,434 PROGRAMS. 902 00:42:42,434 --> 00:42:43,969 SO WE THINK THAT A LOT OF THIS 903 00:42:43,969 --> 00:42:45,738 HOPEFULLY WILL CONVERGE AND COME 904 00:42:45,738 --> 00:42:47,473 TOGETHER AS WE LEVERAGE THESE 905 00:42:47,473 --> 00:42:51,744 VARIOUS RESOURCES, THE MOLECULAR 906 00:42:51,744 --> 00:42:54,046 ATLASES LIKE LUNG MAP, THE 907 00:42:54,046 --> 00:42:58,417 TISSUE REPOSITORIES, ALONG WITH 908 00:42:58,417 --> 00:42:59,785 TOPMED AND THE APPLICATION OF 909 00:42:59,785 --> 00:43:01,153 THE RADIOMICS OF IMAGING 910 00:43:01,153 --> 00:43:04,890 TECHNOLOGIES WE HAVE IN BDC, ALL 911 00:43:04,890 --> 00:43:09,495 DRIVEN IN AN AI/ML ANALYTICS 912 00:43:09,495 --> 00:43:11,130 THAT'S REALLY COME TOGETHER IN 913 00:43:11,130 --> 00:43:15,067 WAYS OF INTEGRATING ALL THIS 914 00:43:15,067 --> 00:43:16,168 INFORMATION. 915 00:43:16,168 --> 00:43:18,137 PRESUMABLY AND HOPEFULLY IN A 916 00:43:18,137 --> 00:43:20,873 PRE-CLINICAL CONTEXT TO REALLY 917 00:43:20,873 --> 00:43:23,275 MOVE THIS AREA FORWARD IN A WAY 918 00:43:23,275 --> 00:43:25,144 THAT SERVES THESE PATIENTS MUCH 919 00:43:25,144 --> 00:43:30,482 BETTER. 920 00:43:30,482 --> 00:43:32,017 SO I JUST TRIED TO OUTLINE AT 921 00:43:32,017 --> 00:43:35,087 LEAST A COUPLE OF EMERGING 922 00:43:35,087 --> 00:43:36,522 OPPORTUNITIES THAT FRANKLY I 923 00:43:36,522 --> 00:43:37,723 HOPE WE CAN START TO MOVE ON IN 924 00:43:37,723 --> 00:43:40,793 THE NEXT YEAR OR TWO RELATED TO 925 00:43:40,793 --> 00:43:45,731 WOMEN'S HEALTH, AND CHRONIC LUNG 926 00:43:45,731 --> 00:43:48,133 DISEASE AND MOLECULAR 927 00:43:48,133 --> 00:43:49,768 CHARACTERIZATION, RADIOMICS, 928 00:43:49,768 --> 00:43:52,070 LEVERAGING OUR TOP MED RESOURCES 929 00:43:52,070 --> 00:43:55,374 AND OUR BIODATA CATALYSTS, 930 00:43:55,374 --> 00:43:57,776 PARTNERING WITH DATA SCIENTISTS 931 00:43:57,776 --> 00:44:02,281 CAN HELP US APPLY AI/ML 932 00:44:02,281 --> 00:44:06,652 GENERATED TOOLS ALONG WITH 933 00:44:06,652 --> 00:44:08,854 DISCOVERY SCIENTISTS IN HEART, 934 00:44:08,854 --> 00:44:09,822 LUNG AND BLOOD SLEEP SCIENCE IN 935 00:44:09,822 --> 00:44:11,690 WAYS THAT WE THINK COULD HAVE A 936 00:44:11,690 --> 00:44:12,991 DRAMATIC IMPACT FOR BOTH THOSE 937 00:44:12,991 --> 00:44:15,861 TWO DOMAINS OF WOMEN'S HEALTH. 938 00:44:15,861 --> 00:44:16,195 IN 939 00:44:16,195 --> 00:44:17,196 CHRONIC LUNG DISEASE. 940 00:44:17,196 --> 00:44:21,333 AND WITH THAT, I WILL SIT DOWN. 941 00:44:21,333 --> 00:44:22,534 SO THANK YOU VERY MUCH FOR YOUR 942 00:44:22,534 --> 00:44:25,604 ATTENTION. 943 00:44:25,604 --> 00:44:32,878 [APPLAUSE] 944 00:44:32,878 --> 00:44:34,413 >> THANK YOU VERY MUCH, 945 00:44:34,413 --> 00:44:35,214 DR. GIBBONS. 946 00:44:35,214 --> 00:44:37,015 DOES ANYONE HAVE ANY QUESTIONS, 947 00:44:37,015 --> 00:44:46,758 COMMENTS FOR DR. G GIBBONS? 948 00:44:46,758 --> 00:44:48,093 HEARING NONE, OUR NEXT SPEAKER 949 00:44:48,093 --> 00:44:51,797 IS DR. COURTNEY FITZHUGH. 950 00:44:51,797 --> 00:44:57,703 SHE RECEIVED HER B.S. MAGNA CUM 951 00:44:57,703 --> 00:45:00,105 LAUDE FROM LOS ANGELES IN 952 00:45:00,105 --> 00:45:00,339 1996 -- 953 00:45:00,339 --> 00:45:02,975 >> CAN I -- HOLD ON, COURTNEY? 954 00:45:02,975 --> 00:45:07,679 IF YOU DON'T MIND, I'D LIKE TO 955 00:45:07,679 --> 00:45:11,717 MAKE A PROVIZATIONAL 956 00:45:11,717 --> 00:45:13,252 INTERJECTION HERE, I THINK I 957 00:45:13,252 --> 00:45:14,019 HAVE THAT PREROGATIVE. 958 00:45:14,019 --> 00:45:16,755 WE'LL GET TO YOU, COURTNEY. 959 00:45:16,755 --> 00:45:19,291 AS AN EXTENSION OF OUR 960 00:45:19,291 --> 00:45:21,727 ACKNOWLEDGMENT OF DR. KILEY'S 40 961 00:45:21,727 --> 00:45:26,698 YEARS OF SERVICE, DR. CHILDS, 962 00:45:26,698 --> 00:45:29,434 WOULD YOU BE SO KIND AS TO COME 963 00:45:29,434 --> 00:45:32,838 TO THE FRONT OF THE PODIUM AND 964 00:45:32,838 --> 00:45:38,744 DO THE HONORS O TO DESCRIBE WHAT 965 00:45:38,744 --> 00:45:40,379 YOU'D LIKE DR. KILEY TO ENGAGE 966 00:45:40,379 --> 00:45:41,380 IN? 967 00:45:41,380 --> 00:45:46,318 [LAUGHTER] 968 00:45:46,318 --> 00:45:48,287 >> I'VE HEARD DR. KILEY LOVES 969 00:45:48,287 --> 00:45:50,022 SURPRISES. 970 00:45:50,022 --> 00:45:52,324 SO HE'S GOING TO LOVE THIS. 971 00:45:52,324 --> 00:45:54,626 BUT -- SO I'M A MEMBER OF THE 972 00:45:54,626 --> 00:45:57,696 UNIFORMED SERVICE, THERE'S EIGHT 973 00:45:57,696 --> 00:46:00,732 UNIFORMED SERVICES, ARMY, NAVY, 974 00:46:00,732 --> 00:46:03,702 AIR FORCE, MARINES, PEACE CORPS, 975 00:46:03,702 --> 00:46:04,670 NOAA AND THE PUBLIC HEALTH 976 00:46:04,670 --> 00:46:05,570 SERVICE. 977 00:46:05,570 --> 00:46:08,206 ONE OF TWO SERVICES THAT WE 978 00:46:08,206 --> 00:46:09,942 DON'T CARRY GUNS. 979 00:46:09,942 --> 00:46:11,310 WE CARRY STETHOSCOPES IN THE 980 00:46:11,310 --> 00:46:14,880 PUBLIC HEALTH SERVICE. 981 00:46:14,880 --> 00:46:18,684 AND ONE OF OUR TRADITIONS IS 982 00:46:18,684 --> 00:46:21,653 THAT WHEN YOU HIT A BENCHMARK 983 00:46:21,653 --> 00:46:24,723 THAT'S IMPORTANT AND NOTEWORTHY, 984 00:46:24,723 --> 00:46:27,893 YOU MAY BE A CANDIDATE FOR 985 00:46:27,893 --> 00:46:31,396 GETTING A COIN. 986 00:46:31,396 --> 00:46:33,999 AND IT'S HARD TO HIT THAT 987 00:46:33,999 --> 00:46:34,833 BENCHMARK. 988 00:46:34,833 --> 00:46:36,101 SOMETIMES GARY AND I WERE 989 00:46:36,101 --> 00:46:37,936 TALKING AND HE'S LIKE, IS THAT 990 00:46:37,936 --> 00:46:38,704 COIN-WORTHY? 991 00:46:38,704 --> 00:46:41,106 I'M LIKE, NO, GARY, THAT'S NOT 992 00:46:41,106 --> 00:46:41,440 COIN-WORTHY. 993 00:46:41,440 --> 00:46:43,508 BUT I JUST WANT TO -- ON BEHALF 994 00:46:43,508 --> 00:46:46,144 OF THE UNITED STATES PUBL PUBLIC 995 00:46:46,144 --> 00:46:47,012 HEALTH SERVICE, RECOGNIZE 996 00:46:47,012 --> 00:46:49,314 DR. KILEY FOR ALL HIS WORK THAT 997 00:46:49,314 --> 00:46:54,252 HAS IMPACTED PUBLIC HEALTH AS IT 998 00:46:54,252 --> 00:46:58,056 APPLIES TO PULMONARY DISEASE AND 999 00:46:58,056 --> 00:47:02,995 RECOGNIZE HIM BY GIVING HIM MY 1000 00:47:02,995 --> 00:47:06,598 CHALLENGE COIN, WHICH HAS ON 1001 00:47:06,598 --> 00:47:09,234 IT -- HOPEFULLY HE CAN SHOW YOU 1002 00:47:09,234 --> 00:47:14,373 LATER -- HEART, LUNG AND BLOOD. 1003 00:47:14,373 --> 00:47:15,574 HE MIGHT BE A LITTLE 1004 00:47:15,574 --> 00:47:17,676 DISAPPOINTED THAT IT'S NOT LUNG, 1005 00:47:17,676 --> 00:47:19,311 HEART AND BLOOD, BUT I WANT TO 1006 00:47:19,311 --> 00:47:21,380 PRESENT THIS TO YOU TODAY IN 1007 00:47:21,380 --> 00:47:22,814 RECOGNITION. 1008 00:47:22,814 --> 00:47:24,383 THIS IS -- OF ALL THE WORK THAT 1009 00:47:24,383 --> 00:47:25,350 HE'S DONE. 1010 00:47:25,350 --> 00:47:35,527 [APPLAUSE] 1011 00:47:37,029 --> 00:47:37,696 >> THANK YOU SO MUCH. 1012 00:47:37,696 --> 00:47:45,604 >> YOU'RE WELCOME. 1013 00:47:45,604 --> 00:47:47,339 >> THANK YOU ALL VERY MUCH. 1014 00:47:47,339 --> 00:47:48,673 SUCH A SPECIAL RECOGNITION, 1015 00:47:48,673 --> 00:47:51,843 PARTICULARLY FROM MY COLLEAGUES. 1016 00:47:51,843 --> 00:47:54,679 A CARRIES A HUGE AMOUNT OF 1017 00:47:54,679 --> 00:47:57,682 WEIGHT, SO IT'S REALLY WONDERFUL 1018 00:47:57,682 --> 00:47:59,317 TO BE RECOGNIZED. 1019 00:47:59,317 --> 00:48:08,293 [APPLAUSE] 1020 00:48:08,293 --> 00:48:11,329 >> HEY, JIM, HOLD ON A SECOND. 1021 00:48:11,329 --> 00:48:15,600 RICK, DOES THIS REQUIRE A VOTE? 1022 00:48:15,600 --> 00:48:17,436 >> YOU'LL ALL HAVE TO RECUSE 1023 00:48:17,436 --> 00:48:19,771 YOURSELVES. 1024 00:48:19,771 --> 00:48:24,142 >> I THINK IF JIM JUST TAKES THE 1025 00:48:24,142 --> 00:48:29,614 COIN AND MOVES IT, IT COULD B 1026 00:48:29,614 --> 00:48:30,582 BE -- 1027 00:48:30,582 --> 00:48:33,985 [LAUGHTER] 1028 00:48:33,985 --> 00:48:36,588 >> BACK TO THE AGENDA. 1029 00:48:36,588 --> 00:48:39,758 WE HAVE DR. COURTNEY FITZHUGH TO 1030 00:48:39,758 --> 00:48:50,669 SPEAK TO US ABOUT HEMATOPOI -- E 1031 00:48:51,536 --> 00:48:52,571 PARTICIPATED IN THE NIH CLINICAL 1032 00:48:52,571 --> 00:48:53,572 RESEARCH TRAINING PROGRAM, WHERE 1033 00:48:53,572 --> 00:48:56,842 SHE STUDIED WITH DR. JOHN 1034 00:48:56,842 --> 00:49:01,113 TISDALE IN NIDDK AND NHLBI AFTER 1035 00:49:01,113 --> 00:49:03,081 RECEIVING HER M.D., DR. FITZHUGH 1036 00:49:03,081 --> 00:49:05,484 COMPLETED A JOINT RESIDENCY IN 1037 00:49:05,484 --> 00:49:09,221 INTERNAL MEDICINE AND PEDIATRICS 1038 00:49:09,221 --> 00:49:10,088 AT DUKE UNIVERSITY MEDICAL 1039 00:49:10,088 --> 00:49:13,725 CENTER AND IN 2005 SHE DID A 1040 00:49:13,725 --> 00:49:16,995 COMBINED HEMATOLOGY ONCOLOGY 1041 00:49:16,995 --> 00:49:18,530 FELLOWSHIP AT THE NIH AND JOHNS 1042 00:49:18,530 --> 00:49:21,099 HOPKINS HOSPITAL. 1043 00:49:21,099 --> 00:49:22,801 IN 2007, DR. FITZHUGH RETURNED 1044 00:49:22,801 --> 00:49:26,104 TO THE NHLBI AND WAS APPOINTED 1045 00:49:26,104 --> 00:49:28,273 AS ASSISTANT CLINICAL 1046 00:49:28,273 --> 00:49:31,576 INVESTIGATOR IN 2012 AND LASKER 1047 00:49:31,576 --> 00:49:34,513 CLINICAL RESEARCH SCHOLAR AS A 1048 00:49:34,513 --> 00:49:35,847 CLINICAL TENURE TRACK 1049 00:49:35,847 --> 00:49:37,048 INVESTIGATOR IN 2016. 1050 00:49:37,048 --> 00:49:38,817 HER CURRENT RESEARCH INCLUDES 1051 00:49:38,817 --> 00:49:41,553 EXPLORING NEW AVENUES OF 1052 00:49:41,553 --> 00:49:42,754 HEMATOPOIETIC CELL 1053 00:49:42,754 --> 00:49:46,458 TRANSPLANTATION FOR SICKLE CELL 1054 00:49:46,458 --> 00:49:51,296 DISEASE, AND STUDYING THE LONG 1055 00:49:51,296 --> 00:49:53,698 TERM HEALTH EFFECTS OF KERATIN 1056 00:49:53,698 --> 00:49:54,332 THERAPIES FOR SICKLE CELL 1057 00:49:54,332 --> 00:49:54,799 DISEASE. 1058 00:49:54,799 --> 00:49:57,002 HER TEAM HAS BEEN DEVELOPING AN 1059 00:49:57,002 --> 00:49:58,203 ALTERNATIVE APPROACH WHERE 1060 00:49:58,203 --> 00:50:00,038 PATIENTS RECEIVE AGENTS FOCUSED 1061 00:50:00,038 --> 00:50:00,939 OND ERADICATING THE IMMUNE 1062 00:50:00,939 --> 00:50:02,307 SYSTEM AND MAKING SPACE IN THE 1063 00:50:02,307 --> 00:50:04,576 BONE MARROW WHILE AVOIDING HIGH 1064 00:50:04,576 --> 00:50:05,544 DOSE CHEMOTHERAPY. 1065 00:50:05,544 --> 00:50:07,179 SHE HAS DEMONSTRATED THE 1066 00:50:07,179 --> 00:50:10,916 EFFICACY OF THIS 1067 00:50:10,916 --> 00:50:12,350 NON-MYELOABLATIVE PROCEDURE IN 1068 00:50:12,350 --> 00:50:14,186 BOTH MICE AND HUMAN VOLUNTEERS, 1069 00:50:14,186 --> 00:50:16,521 AND IS CURRENTLY PARTICIPATING 1070 00:50:16,521 --> 00:50:18,490 IN A LONG-TERM FOLLOW-UP STUDY 1071 00:50:18,490 --> 00:50:19,791 WITH PATIENTS TO SEE IF THEY 1072 00:50:19,791 --> 00:50:20,458 REMAIN FREE OF SICKLE CELL 1073 00:50:20,458 --> 00:50:23,195 DISEASE. 1074 00:50:23,195 --> 00:50:24,062 ADDITIONALLY, SHE IS ALSO 1075 00:50:24,062 --> 00:50:25,463 INTERESTED IN LEARNING MORE 1076 00:50:25,463 --> 00:50:26,731 ABOUT THE LONG TERM HEALTH 1077 00:50:26,731 --> 00:50:29,134 EFFECTS OF CURATIVE THERAPIES IN 1078 00:50:29,134 --> 00:50:29,768 PATIENTS WITH SICKLE CELL 1079 00:50:29,768 --> 00:50:30,435 DISEASE. 1080 00:50:30,435 --> 00:50:32,070 SHE'S A MEMBER OF THE AMERICAN 1081 00:50:32,070 --> 00:50:34,105 SOCIETY OF HEMATOLOGY AND THE 1082 00:50:34,105 --> 00:50:35,173 AMERICAN SOCIETY OF 1083 00:50:35,173 --> 00:50:37,542 TRANSPLANTATION AND CELLULAR 1084 00:50:37,542 --> 00:50:37,876 THERAPY. 1085 00:50:37,876 --> 00:50:39,978 AND MOST RECENTLY SHE WAS ONE OF 1086 00:50:39,978 --> 00:50:41,413 ONLY SEVEN NIH INVESTIGATORS 1087 00:50:41,413 --> 00:50:43,148 ELECTED TO THE AMERICAN SOCIETY 1088 00:50:43,148 --> 00:50:46,651 FOR CLINICAL INVESTIGATION. 1089 00:50:46,651 --> 00:50:47,752 THIS YEAR. 1090 00:50:47,752 --> 00:50:49,955 WELCOME, DR. FITZHUGH. 1091 00:50:49,955 --> 00:50:51,389 >> THANK YOU SO MUCH FOR THE -- 1092 00:50:51,389 --> 00:50:56,528 [APPLAUSE] 1093 00:50:56,528 --> 00:50:57,729 >> THANK YOU FOR THE 1094 00:50:57,729 --> 00:50:58,730 INTRODUCTION AND FOR INVITING ME 1095 00:50:58,730 --> 00:51:00,031 TO GIVE THIS PRESENTATION. 1096 00:51:00,031 --> 00:51:02,767 SO MY TALK IS ENTITLED CLONAL 1097 00:51:02,767 --> 00:51:03,702 HEMATOPOE CYST FOLLOWING 1098 00:51:03,702 --> 00:51:04,636 CURATIVE THERAPIES FOR SICKLE 1099 00:51:04,636 --> 00:51:07,339 CELL DISEASE. 1100 00:51:07,339 --> 00:51:08,506 I'M GOING TO START BY 1101 00:51:08,506 --> 00:51:09,474 INTRODUCING YOU TO ONE OF MY 1102 00:51:09,474 --> 00:51:10,375 PATIENTS I'M GOING TO REFER TO 1103 00:51:10,375 --> 00:51:13,111 AS MR. SMITH. 1104 00:51:13,111 --> 00:51:15,714 HE WAS A 36-YEAR-OLD AFRICAN 1105 00:51:15,714 --> 00:51:17,582 AMERICAN GENTLEMAN WITH SICKLE 1106 00:51:17,582 --> 00:51:24,689 CELL DISEASE. 1107 00:51:24,689 --> 00:51:27,692 HE ALSO HAD CHRONIC RENAL 1108 00:51:27,692 --> 00:51:29,861 INSUFFICIENCY WITH BASELINE 1109 00:51:29,861 --> 00:51:36,301 CREATININE OF 2.5 MG PER DL. 1110 00:51:36,301 --> 00:51:39,271 SICKLE CELL DISEASE IS CAUSED BY 1111 00:51:39,271 --> 00:51:40,272 MUTATION THAT LEADS TO THE 1112 00:51:40,272 --> 00:51:42,574 TRANSFORMATION OF RED BLOOD 1113 00:51:42,574 --> 00:51:43,975 CELLS FROM CONCAVE -- THAT 1114 00:51:43,975 --> 00:51:46,077 EASILY MOVE THROUGHOUT THE MICRO 1115 00:51:46,077 --> 00:51:50,448 VASCULATURE TO A RIGID -- 1116 00:51:50,448 --> 00:51:51,716 ASSOCIATED WITH DAMAGE TO THE 1117 00:51:51,716 --> 00:51:57,355 BONE MARROW MICROENVIRONMENT, 1118 00:51:57,355 --> 00:51:58,657 INFLAMMATION AND OXIDATIVE 1119 00:51:58,657 --> 00:52:00,558 STRESS. 1120 00:52:00,558 --> 00:52:06,231 IN THIS FIGURE YOU SEE THE -- 1121 00:52:06,231 --> 00:52:07,899 SURVIVAL -- THESE ARE IN 25 1122 00:52:07,899 --> 00:52:09,834 PATIENTS MOSTLY WITH HOMOZYGOUS 1123 00:52:09,834 --> 00:52:12,170 SICKLE CELL DISEASE MEDIAN OF 1124 00:52:12,170 --> 00:52:12,837 6 1/2 YEARS. 1125 00:52:12,837 --> 00:52:16,875 YOU SEE 60% OF THE PATIENTS WERE 1126 00:52:16,875 --> 00:52:19,944 TAKEN -- HYDROXYUREA -- AT LEAST 1127 00:52:19,944 --> 00:52:21,279 20 YEARS EARLIER THAN THE 1128 00:52:21,279 --> 00:52:21,913 GENERAL AFRICAN AMERICAN 1129 00:52:21,913 --> 00:52:31,756 POPULATION. 1130 00:52:31,756 --> 00:52:33,325 -- WANTED TO KNOW WHETHER 1131 00:52:33,325 --> 00:52:35,827 HAVING MORE THAN ONE ORGAN 1132 00:52:35,827 --> 00:52:38,229 IMPAIRED LEADS TO FURTHER 1133 00:52:38,229 --> 00:52:42,901 INCREASE IN MORTALITY. 1134 00:52:42,901 --> 00:52:45,203 LUNG IMPAIRMENT LESS THAN 70% 1135 00:52:45,203 --> 00:52:47,739 AND KIDNEY IMPAIRMENT HAD 1136 00:52:47,739 --> 00:52:49,474 MULTIPLE DEFINITIONS INCLUDING 1137 00:52:49,474 --> 00:52:51,209 ESTIMATED GFR OF LESS THAN 60. 1138 00:52:51,209 --> 00:52:53,411 YOU SEE PATIENTS WHO HAD ZERO OR 1139 00:52:53,411 --> 00:52:54,412 ONE ORGAN IMPAIRED HAD A 1140 00:52:54,412 --> 00:52:56,381 MEANTIME TO DEATH OF 14 YEARS, 1141 00:52:56,381 --> 00:52:57,582 WHILE PATIENTS WITH MORE THAN 1142 00:52:57,582 --> 00:52:58,783 ONE ORGAN IMPAIRED HAD A 1143 00:52:58,783 --> 00:53:00,885 MEANTIME TO DEATH OF ONLY 1144 00:53:00,885 --> 00:53:01,653 7.8 YEARS. 1145 00:53:01,653 --> 00:53:03,521 AND BECAUSE MR. SMITH HAD BOTH 1146 00:53:03,521 --> 00:53:06,257 HEART AND KIDNEY IMPAIRMENT, HIS 1147 00:53:06,257 --> 00:53:07,792 RISK OF EARLY MORTALITY WAS EVEN 1148 00:53:07,792 --> 00:53:12,397 GREATER. 1149 00:53:12,397 --> 00:53:13,031 PATIENTS WITH SICKLE CELL 1150 00:53:13,031 --> 00:53:15,700 DISEASE HAVE INCREASED RISK FOR 1151 00:53:15,700 --> 00:53:23,007 AGGRESSIVE MYELOID LIG M MALIGN. 1152 00:53:23,007 --> 00:53:25,009 IDENTIFIED BETWEEN 1991 AND 1153 00:53:25,009 --> 00:53:27,746 2014, AND THEY WERE FOLLOWED FOR 1154 00:53:27,746 --> 00:53:28,713 OVER 140,000 PERSON YEARS AND 1155 00:53:28,713 --> 00:53:30,482 COMPARED TO THE GENERAL 1156 00:53:30,482 --> 00:53:30,915 POPULATION. 1157 00:53:30,915 --> 00:53:38,056 THEY DID NOT FIND AN INCREA 1158 00:53:38,056 --> 00:53:40,125 INCREASED -- 3.6 FOLD 1159 00:53:40,125 --> 00:53:43,762 INCREASE -- OR AML. 1160 00:53:43,762 --> 00:53:44,729 THEY ALSO FOUND PATIENTS WITH 1161 00:53:44,729 --> 00:53:45,630 SICKLE CELL DISEASE WHO ARE 1162 00:53:45,630 --> 00:53:47,165 OLDER AND THOSE WITH MORE SEVERE 1163 00:53:47,165 --> 00:53:49,467 DISEASE HAD A HIGHER RISK OF 1164 00:53:49,467 --> 00:53:50,769 LEUKEMIA, BUT THEY ALSO COMPARED 1165 00:53:50,769 --> 00:53:52,537 THE PREVALENCE OF LEUKEMIA 1166 00:53:52,537 --> 00:53:55,940 BEFORE AND AFTER HYDROXYUREA WAS 1167 00:53:55,940 --> 00:53:57,475 FDA-APPROVED IN 1998 AND THEY 1168 00:53:57,475 --> 00:53:58,777 DID NOT FIND INCREASED 1169 00:53:58,777 --> 00:54:02,180 PREVALENCE OF LEUKEMIA AFTER 1170 00:54:02,180 --> 00:54:03,281 HYDROXYUREA WAS FDA-APPROVED. 1171 00:54:03,281 --> 00:54:05,049 IF YOU LOOK CLOSELY AT THE DATA, 1172 00:54:05,049 --> 00:54:06,785 YOU SEE AGAIN THAT OVER 6400 1173 00:54:06,785 --> 00:54:07,452 PATIENTS WITH SICKLE CELL 1174 00:54:07,452 --> 00:54:09,287 DISEASE WERE FOLLOWED FOR OVER 1175 00:54:09,287 --> 00:54:10,522 27 YEARS. 1176 00:54:10,522 --> 00:54:12,123 DURING THAT TIME, ONLY SIX 1177 00:54:12,123 --> 00:54:14,793 PATIENTS DEVELOPED AML WITH OVER 1178 00:54:14,793 --> 00:54:16,227 140,000 PERSON YEARS OF 1179 00:54:16,227 --> 00:54:19,397 FOLLOW-UP, OR 0.004 PER 100 1180 00:54:19,397 --> 00:54:19,964 PERSON YEARS. 1181 00:54:19,964 --> 00:54:22,567 THIS COMPARES TO IS .67 1182 00:54:22,567 --> 00:54:24,335 INDIVIDUALS WITHOUT SICKLE CELL 1183 00:54:24,335 --> 00:54:25,470 DISEASE WHO ARE EXPECTED TO 1184 00:54:25,470 --> 00:54:28,706 DEVELOP AML OVER THAT TIME FRAME 1185 00:54:28,706 --> 00:54:30,008 WHEN CONTROLLED FOR AGE, SEX, 1186 00:54:30,008 --> 00:54:34,846 RACE AND ETHNICITY OR 0.001 PER 1187 00:54:34,846 --> 00:54:35,280 100 PERSON-YEARS. 1188 00:54:35,280 --> 00:54:36,614 SO WHILE THERE'S AN INCREASED 1189 00:54:36,614 --> 00:54:37,916 RELATIVE RISK FOR LEUKEMIA IN 1190 00:54:37,916 --> 00:54:39,451 PATIENTS WITH SICKLE CELL 1191 00:54:39,451 --> 00:54:41,319 DISEASE OR AML SPECIFICALLY, THE 1192 00:54:41,319 --> 00:54:43,788 ABSOLUTE RISK IS LOW. 1193 00:54:43,788 --> 00:54:46,858 THERE ARE MULTIPLE RISK FACTORS 1194 00:54:46,858 --> 00:54:51,229 FOR MYELOID -- INFLAMMATION, 1195 00:54:51,229 --> 00:54:54,299 HYPOXIA AND ACIDOSIS, 1196 00:54:54,299 --> 00:54:55,200 TRANSFUSION-RELATED 1197 00:54:55,200 --> 00:54:57,302 IMMUNOMODULATION, COMPROMISED 1198 00:54:57,302 --> 00:54:58,837 APOPTOSIS, ENDOTHELIAL DAMAGE, 1199 00:54:58,837 --> 00:55:04,742 INCREASED AGE, AND CLONAL 1200 00:55:04,742 --> 00:55:06,377 HEMATOPOESIS. 1201 00:55:06,377 --> 00:55:11,115 -- OFFERS A POTENTIAL CURE FOR 1202 00:55:11,115 --> 00:55:11,850 PATIENT WITH SICKLE CELL 1203 00:55:11,850 --> 00:55:12,083 DISEASE. 1204 00:55:12,083 --> 00:55:13,718 IN ORDER TO COMPLETELY WIPE OUT 1205 00:55:13,718 --> 00:55:16,087 THEIR BONE MAY OTHER AND REPLACE 1206 00:55:16,087 --> 00:55:26,531 IT WITH THAT OF THE DONOR. 1207 00:55:28,266 --> 00:55:29,734 93% OF THE PATIENTS WERE ALIVE, 1208 00:55:29,734 --> 00:55:32,470 AND EVENT-FREE SURVIVAL OF 91%, 1209 00:55:32,470 --> 00:55:34,138 MEANING AT FIVE YEARS, 91% WERE 1210 00:55:34,138 --> 00:55:35,139 ALIVE AND FREE OF SICKLE CELL 1211 00:55:35,139 --> 00:55:37,141 DISEASE. 1212 00:55:37,141 --> 00:55:39,010 THE FIVE-YEAR OVERALL SURVIVAL 1213 00:55:39,010 --> 00:55:40,745 AND EVENT-FREE SURVIVAL WERE A 1214 00:55:40,745 --> 00:55:43,081 LITTLE HIGHER AN 95 AND 93% 1215 00:55:43,081 --> 00:55:43,748 RESPECTIVELY FOR PATIENTS 1216 00:55:43,748 --> 00:55:45,483 YOUNGER THAN 16 YEARS OF AGE, 1217 00:55:45,483 --> 00:55:46,818 AND THAT WASN'T SURPRISING SINCE 1218 00:55:46,818 --> 00:55:48,786 MANY OF THE PATIENTS RECEIVED 1219 00:55:48,786 --> 00:55:49,554 MYELOABLATIVE DOSES OF 1220 00:55:49,554 --> 00:55:52,190 CHEMOTHERAPY. 1221 00:55:52,190 --> 00:55:54,259 THEY ALSO REPORT THE A 1222 00:55:54,259 --> 00:55:57,028 CUMULATIVE INCIDENCE OF 1223 00:55:57,028 --> 00:55:59,531 GRAFT-VERSUS-HOST DISEASE OF 15% 1224 00:55:59,531 --> 00:56:02,800 AND CHRONIC OF 14%. 1225 00:56:02,800 --> 00:56:06,237 IT'S WHEN IT RECOGNIZES IT AS 1226 00:56:06,237 --> 00:56:09,841 BEING FOREIGN AND ATTACKS THE 1227 00:56:09,841 --> 00:56:13,912 PATIENT, CAN BE DEADLY, SO YOU D 1228 00:56:13,912 --> 00:56:22,554 BECAUSE THERE'S NO BENEFIT -- SO 1229 00:56:22,554 --> 00:56:24,756 THE FIRST MAJOR OBSTACLE IS THAT 1230 00:56:24,756 --> 00:56:25,723 MANY ADULTS WITH SICKLE CELL 1231 00:56:25,723 --> 00:56:27,225 DISEASE WHO RECEIVE THE STANDARD 1232 00:56:27,225 --> 00:56:29,827 CURATIVE THERAPY WHICH INCLUDES 1233 00:56:29,827 --> 00:56:31,329 MYELOABLATIVE CONDITIONING WILL 1234 00:56:31,329 --> 00:56:31,796 DIE. 1235 00:56:31,796 --> 00:56:35,733 WE WANTED TO DEVELOP A NON-MILE 1236 00:56:35,733 --> 00:56:36,734 MYELOABLATIVE APPROACH WHERE 1237 00:56:36,734 --> 00:56:38,803 INSTEAD OF GIVING HIGH DOSE 1238 00:56:38,803 --> 00:56:40,138 CHEMOTHERAPY WE WOULD SUPPRESS 1239 00:56:40,138 --> 00:56:41,806 THE IMMUNE SYSTEM IN ORDER TO 1240 00:56:41,806 --> 00:56:46,945 REDUCE THE RISK FOR GRAFT 1241 00:56:46,945 --> 00:56:48,913 REJECTION OR GRAFT-VERSUS-HOST 1242 00:56:48,913 --> 00:56:50,648 DISEASE. 1243 00:56:50,648 --> 00:57:01,292 PATIENTS RECEIVE O ALEMTUZUMAB. 1244 00:57:01,926 --> 00:57:03,528 -- ALSO MAKES SPACE FOR THE 1245 00:57:03,528 --> 00:57:04,495 DONOR CELLS. 1246 00:57:04,495 --> 00:57:09,534 AND WE GIVE SIROLIMUS, SO THE 1247 00:57:09,534 --> 00:57:11,869 DONOR AND RECIPIENT CELLS NO 1248 00:57:11,869 --> 00:57:13,504 LONGER RECOGNIZE THIS THING AS 1249 00:57:13,504 --> 00:57:14,272 FOREIGN. 1250 00:57:14,272 --> 00:57:17,575 SO NIH AND COLLEAGUES AT THE 1251 00:57:17,575 --> 00:57:19,310 UNIVERSITY OF ILLINOIS AT 1252 00:57:19,310 --> 00:57:22,013 CHICAGO RECENTLY DESCRIBED 1253 00:57:22,013 --> 00:57:23,481 PATIENTS USING THIS APROA. THE 1254 00:57:23,481 --> 00:57:25,817 MEDIAN FOLLOW-UP WAS FOUR YEARS 1255 00:57:25,817 --> 00:57:28,453 AND THE OVERALL SURVIVAL WAS 1256 00:57:28,453 --> 00:57:30,855 93%, THE EVENT-FREE SURVIVAL WAS 1257 00:57:30,855 --> 00:57:31,055 85%. 1258 00:57:31,055 --> 00:57:36,227 NONE OF THE PATIENTS HAD GRADE D 1259 00:57:36,227 --> 00:57:38,529 CHRONIC GVHD. 1260 00:57:38,529 --> 00:57:41,466 THE SECOND MAJOR OBSTACLE FROM 1261 00:57:41,466 --> 00:57:43,468 CURING MR. SMITH IS -- OF 1262 00:57:43,468 --> 00:57:44,102 PATIENTS WITH SICKLE CELL 1263 00:57:44,102 --> 00:57:46,638 DISEASE HAVE A FULLY HLA 1264 00:57:46,638 --> 00:57:47,305 MAST-RELATED DONOR. 1265 00:57:47,305 --> 00:57:49,641 ON THE OTHER HAND, 90% OR AT 1266 00:57:49,641 --> 00:57:51,042 LEAST 90% OF PATIENTS WILL HAVE 1267 00:57:51,042 --> 00:57:56,881 A PARENT, A CHILD OR -- MATCHED 1268 00:57:56,881 --> 00:57:59,617 SIBLING -- THIS GREATLY EXPANDS 1269 00:57:59,617 --> 00:58:01,786 THE DONOR POOL, ALLOWING 1270 00:58:01,786 --> 00:58:03,021 ENROLLMENT OF PATIENTS WHO HAVE 1271 00:58:03,021 --> 00:58:04,122 ORGAN DAMAGE. 1272 00:58:04,122 --> 00:58:08,292 SO NON-MYELOABLATIVE 1273 00:58:08,292 --> 00:58:08,826 HAPLOIDENTICAL TRANSPLANT 1274 00:58:08,826 --> 00:58:09,961 EXPANDS THE DONOR POOL AND 1275 00:58:09,961 --> 00:58:11,496 ALLOWS PATIENTS WITH 1276 00:58:11,496 --> 00:58:13,898 COMORBIDITIES TO UNDER GS 1277 00:58:13,898 --> 00:58:14,198 TRANSPLANT. 1278 00:58:14,198 --> 00:58:15,900 WHEN THE ORIGINAL PROTOCOL WAS 1279 00:58:15,900 --> 00:58:17,301 WRITTEN, ONLY THE NIH AND 1280 00:58:17,301 --> 00:58:19,070 HOPKINS WERE PERFORMING 1281 00:58:19,070 --> 00:58:22,473 HAPLOIDENTICAL TRANS PL TRANSPLN 1282 00:58:22,473 --> 00:58:24,909 ADULTS WITH SICKLE CELL DISEASE 1283 00:58:24,909 --> 00:58:32,550 SO -- WE EFFICACY AND TOXICITY 1284 00:58:32,550 --> 00:58:33,584 ASSOCIATED WITH TRANSPLANT WERE 1285 00:58:33,584 --> 00:58:34,018 NOT KNOWN. 1286 00:58:34,018 --> 00:58:35,853 IT WAS WRITTEN AS A DOSE 1287 00:58:35,853 --> 00:58:39,157 ESCALATION OF POST TRANSPLANT -- 1288 00:58:39,157 --> 00:58:40,158 BASED ON -- DATA. 1289 00:58:40,158 --> 00:58:44,095 YOU SEE THE PATIENTS RECEIVED AL 1290 00:58:44,095 --> 00:58:51,569 ALE MTUZUMAB. 1291 00:58:51,569 --> 00:58:53,304 IN THE FIRST COHORT, PATIENTS 1292 00:58:53,304 --> 00:58:56,941 DIDN'T RECEIVE ANY POST 1293 00:58:56,941 --> 00:59:01,546 TRANSPLANT CYCLOPHOSPHAMIDE. 1294 00:59:01,546 --> 00:59:03,414 -- WE MOVED TO THE SECOND 1295 00:59:03,414 --> 00:59:07,118 COHORT, WHERE PATIENTS RECEIVED 1296 00:59:07,118 --> 00:59:08,786 50MG PER KG. 1297 00:59:08,786 --> 00:59:10,321 WE MOVED TO THE THIRD AND FINAL 1298 00:59:10,321 --> 00:59:12,423 COHORT, WHERE PATIENTS WOULD 1299 00:59:12,423 --> 00:59:15,393 RECEIVE 100MG PER KG IN DIVIDED 1300 00:59:15,393 --> 00:59:17,161 DOSES. 1301 00:59:17,161 --> 00:59:18,896 SO 23 PATIENTS WERE TRANSPLANTED 1302 00:59:18,896 --> 00:59:19,864 ON THIS STUDY. 1303 00:59:19,864 --> 00:59:21,666 THE MEDIAN AGE WAS 36 YEARS WITH 1304 00:59:21,666 --> 00:59:26,370 A RANGE OF 20 TO 506 YEARS. 1305 00:59:26,370 --> 00:59:28,339 20 TO 56 YEARS. 1306 00:59:28,339 --> 00:59:30,575 MOST OF THE PATIENTS HAD 1307 00:59:30,575 --> 00:59:32,677 HOMOZYGOUS SICKLE CELL DISEASE, 1308 00:59:32,677 --> 00:59:36,781 TWO HAD TRANSPLANT DEPENDENT 1309 00:59:36,781 --> 00:59:39,183 THALASSEMIA. 1310 00:59:39,183 --> 00:59:41,719 -- HEART FAILURE, AND PULMONARY 1311 00:59:41,719 --> 00:59:42,620 HYPERTENSION, AND THAT'S BECAUSE 1312 00:59:42,620 --> 00:59:44,455 THE EXPECTED BENEFITS WERE 1313 00:59:44,455 --> 00:59:47,125 THOUGHT TO OUTWEIGH THE RISKS. 1314 00:59:47,125 --> 00:59:48,226 SO THESE ARE THE RESULTS OF THE 1315 00:59:48,226 --> 00:59:48,659 STUDY. 1316 00:59:48,659 --> 00:59:50,528 IN THE FIRST COHORT, PATIENTS 1317 00:59:50,528 --> 00:59:53,297 DIDN'T RECEIVE ANY PT -- THREE 1318 00:59:53,297 --> 00:59:54,532 PATIENTS WERE TRANSPLANTED, ONE 1319 00:59:54,532 --> 00:59:56,901 OF THEM INITIALLY ENGRAFTED BUT 1320 00:59:56,901 --> 00:59:58,669 THEN SHE LOST HER GRAFT AT SEVEN 1321 00:59:58,669 --> 01:00:00,772 MONTHS POST TRANSPLANT. 1322 01:00:00,772 --> 01:00:02,406 SO WE -- MOVED TO THE SECOND 1323 01:00:02,406 --> 01:00:05,109 COHORT, WHERE PATIENTS RECEIVED 1324 01:00:05,109 --> 01:00:07,979 ONE DOSE, 15, EIGHT PATIENTS 1325 01:00:07,979 --> 01:00:10,214 WERE TRANSPLANTED, FIVE OF THEM 1326 01:00:10,214 --> 01:00:12,416 INITIALLY -- OR EIGHT OF THEM 1327 01:00:12,416 --> 01:00:13,184 INITIALLY -- FIVE OF THEM 1328 01:00:13,184 --> 01:00:15,186 INITIALLY ENGRAFTED BUT ONLY TWO 1329 01:00:15,186 --> 01:00:15,953 REMAINED FREE OF SICKLE CELL 1330 01:00:15,953 --> 01:00:17,355 DISEASE. 1331 01:00:17,355 --> 01:00:18,756 SO AGAIN WE MOVED TO THE THIRD 1332 01:00:18,756 --> 01:00:21,559 AND FINAL COHORT, WHERE PATIENTS 1333 01:00:21,559 --> 01:00:23,427 RECEIVED 100MG PER KG. 1334 01:00:23,427 --> 01:00:25,496 12 PATIENTS WERE TRANSPLANTED, 1335 01:00:25,496 --> 01:00:27,832 10 INITIALLY ENGRAFTED BUT ONLY 1336 01:00:27,832 --> 01:00:29,100 SIX REMAIN FREE OF SICKLE CELL 1337 01:00:29,100 --> 01:00:29,333 DISEASE. 1338 01:00:29,333 --> 01:00:33,404 SO WITH THE PT -- THE 1339 01:00:33,404 --> 01:00:35,406 ENGRAFTMENT RATE GOT BETTER AND 1340 01:00:35,406 --> 01:00:37,208 THE EVENT-FREE SURVIVAL IMPROVED 1341 01:00:37,208 --> 01:00:39,010 BUT EVEN WITH THE THIRD COHORT, 1342 01:00:39,010 --> 01:00:40,711 THE REJECTION RATE WAS 1343 01:00:40,711 --> 01:00:43,748 UNACCEPTABLY HIGH AND -- SO 1344 01:00:43,748 --> 01:00:45,183 MR. SMITH UNDERWENT 1345 01:00:45,183 --> 01:00:45,817 HAPLOIDENTICAL TRANSPLANT. 1346 01:00:45,817 --> 01:00:48,152 HIS MOTHER WAS THE DONOR. 1347 01:00:48,152 --> 01:00:49,687 UNFORTUNATELY HE REJECTED HIS 1348 01:00:49,687 --> 01:00:50,688 GRAFT WITH SUBSEQUENT RETURN OF 1349 01:00:50,688 --> 01:00:54,292 HIS SICKLE CELL DISEASE. 1350 01:00:54,292 --> 01:00:58,029 WHY DID MR. SMITH'S ATTEMPTED 1351 01:00:58,029 --> 01:01:01,199 CURE FAIL? 1352 01:01:01,199 --> 01:01:05,136 WITH THE TRANSPLANT -- WHILE 1353 01:01:05,136 --> 01:01:07,038 MINIMIZING GRAFT REJECTION AND 1354 01:01:07,038 --> 01:01:07,271 GVHD. 1355 01:01:07,271 --> 01:01:08,806 WITH THE FIRST PROTOCOL, WE HAD 1356 01:01:08,806 --> 01:01:10,241 A LOT OF GRAFT REJECTION AND 1357 01:01:10,241 --> 01:01:13,311 REALLY NO SIGNIFICANT GVHD, SO 1358 01:01:13,311 --> 01:01:15,046 WE HYPOTHESIZED THAT ADDITIONAL 1359 01:01:15,046 --> 01:01:16,714 UP FRONT IMMUNOSUPPRESSION WOULD 1360 01:01:16,714 --> 01:01:17,849 DECREASE THE GRAFT REJECTION 1361 01:01:17,849 --> 01:01:20,351 RATE WHILE AVOIDING UNACCEPTABLE 1362 01:01:20,351 --> 01:01:22,653 GVHC OR VIRAL DISEASE. 1363 01:01:22,653 --> 01:01:25,423 SO THESE ARE TWO NEWER STUDIES 1364 01:01:25,423 --> 01:01:26,757 WHERE WE ATTEMPTED TO DECREASE 1365 01:01:26,757 --> 01:01:28,593 THE RATE OF GRAFT REJECTION 1366 01:01:28,593 --> 01:01:30,494 ESPECIALLY IN A HAPLOIDENTICAL 1367 01:01:30,494 --> 01:01:32,330 SETTING AND BOTH STUDIES INCLUDE 1368 01:01:32,330 --> 01:01:33,764 UP FRONT PRECONDITIONING TO 1369 01:01:33,764 --> 01:01:35,032 PROVIDE MORE IMMUNOSUPPRESSION 1370 01:01:35,032 --> 01:01:43,875 AND WE CHOSE INTRAVENOUS 1371 01:01:43,875 --> 01:01:47,144 PENTOSTA TIB AND DAILY ORAL 1372 01:01:47,144 --> 01:01:53,284 CYCLOPHOSPHAMIDE FOR 14 DAYS. 1373 01:01:53,284 --> 01:01:55,486 BEFORE MOVING ON I WANT TO 1374 01:01:55,486 --> 01:01:56,787 INTRODUCE AUTOLOGOUS GENE 1375 01:01:56,787 --> 01:01:58,022 THERAPY AS A CURATIVE STRATEGY 1376 01:01:58,022 --> 01:01:59,357 FOR PATIENTS WITH SICKLE CELL 1377 01:01:59,357 --> 01:02:00,191 DISEASE. 1378 01:02:00,191 --> 01:02:02,159 I BORROWED THIS SLIDE, THIS IS 1379 01:02:02,159 --> 01:02:02,927 OUR MULTICENTER STUDY AND YOU 1380 01:02:02,927 --> 01:02:05,830 CAN SEE THAT ALL OF THE PATIENTS 1381 01:02:05,830 --> 01:02:07,231 RECEIVED MYELOABLATIVE 1382 01:02:07,231 --> 01:02:07,999 CONDITIONING. 1383 01:02:07,999 --> 01:02:09,667 PATIENTS WERE DIVIDED INTO THREE 1384 01:02:09,667 --> 01:02:10,568 DIFFERENT GROUPS. 1385 01:02:10,568 --> 01:02:13,671 A PRECOLLECTION -- WAS OPTION 1386 01:02:13,671 --> 01:02:15,840 NAND IN GROUP A PATIENTS AND WAS 1387 01:02:15,840 --> 01:02:17,408 REQUIRED FOR GROUPS B AND C 1388 01:02:17,408 --> 01:02:20,912 PATIENTS. 1389 01:02:20,912 --> 01:02:22,113 THE -- BONE MARROW IN GROUP A 1390 01:02:22,113 --> 01:02:24,649 AND B PATIENTS AND MOBILIZED 1391 01:02:24,649 --> 01:02:25,650 PERIPHERAL BLOOD IN GROUP C 1392 01:02:25,650 --> 01:02:26,083 PATIENTS. 1393 01:02:26,083 --> 01:02:28,386 THE ORIGINAL MANUFACTURING 1394 01:02:28,386 --> 01:02:30,588 PROCESS IS GIVEN TO GROUP A AND 1395 01:02:30,588 --> 01:02:32,590 SOME GROUP B PATIENTS AND A MORE 1396 01:02:32,590 --> 01:02:34,659 REFINED PROCESS FOR THE REST OF 1397 01:02:34,659 --> 01:02:36,193 GROUP B AND ALL OF GROUP C 1398 01:02:36,193 --> 01:02:36,627 PATIENTS. 1399 01:02:36,627 --> 01:02:39,363 AND WITH THESE CHANGES, THE CELL 1400 01:02:39,363 --> 01:02:40,731 DOSE THAT RECEIVED BY PATIENTS 1401 01:02:40,731 --> 01:02:42,133 IN GROUP C WAS HIGHER AND 1402 01:02:42,133 --> 01:02:43,801 THERE'S ALSO A LOWER RATE OF 1403 01:02:43,801 --> 01:02:46,404 GRAFT FAILURE. 1404 01:02:46,404 --> 01:02:49,407 SO A TWO-YEAR POST TRANSPLANT, 1405 01:02:49,407 --> 01:02:52,143 MR. SMITH WAS FOUND TO BE 1406 01:02:52,143 --> 01:02:53,911 NEUTROPENIC. 1407 01:02:53,911 --> 01:02:57,014 -- AND PATHOGENIC TP53 1408 01:02:57,014 --> 01:02:57,448 MUTATION. 1409 01:02:57,448 --> 01:03:02,386 HE WAS TREATED WITH DECITABINE 1410 01:03:02,386 --> 01:03:08,893 FOLLOWED BY AZACYTIDINE. 1411 01:03:08,893 --> 01:03:17,568 -- AT THE NIH BETWEEN SEPTEMBE 1412 01:03:17,568 --> 01:03:18,135 DECEMBER 2020. 1413 01:03:18,135 --> 01:03:20,204 18 PATIENTS RECEIVED HLA MATCHED 1414 01:03:20,204 --> 01:03:22,139 SIBLING TRANSPLANT WITH A MEDIAN 1415 01:03:22,139 --> 01:03:23,574 AGE OF 31 YEARS. 1416 01:03:23,574 --> 01:03:25,476 39 PATIENTS UNDERWENT 1417 01:03:25,476 --> 01:03:26,243 HAPLOIDENTICAL TRANSPLANT WITH A 1418 01:03:26,243 --> 01:03:28,145 MEDIAN AGE OF 32 YEARS AND A 1419 01:03:28,145 --> 01:03:31,048 RANGE OF 19 TO 501 YEARS. 1420 01:03:31,048 --> 01:03:34,819 FIVE PATIENTS DEVELOPED EITHER 1421 01:03:34,819 --> 01:03:38,656 MDS OR AML POST TRANSPLANT. 1422 01:03:38,656 --> 01:03:41,392 ALL FIVE PATIENTS HAD HOMOZYGOUS 1423 01:03:41,392 --> 01:03:42,393 SICKLE CELL DISEASE WITH AGE 1424 01:03:42,393 --> 01:03:43,995 RANGING FROM 20 TO 44 YEARS AT 1425 01:03:43,995 --> 01:03:45,162 THE TIME OF TRANSPLANT. 1426 01:03:45,162 --> 01:03:47,498 TWO OF THE PATIENTS UNDERWENT -- 1427 01:03:47,498 --> 01:03:49,667 THE REMAINDER HAPLOIDENTICAL 1428 01:03:49,667 --> 01:03:53,938 TRANSPLANT. 1429 01:03:53,938 --> 01:03:57,441 ONE PATIENT -- FAILURE RANGED 1430 01:03:57,441 --> 01:04:01,746 FROM 73 DAYS TO 7 MONTHS POST 1431 01:04:01,746 --> 01:04:02,747 TRANSPLANT. 1432 01:04:02,747 --> 01:04:04,715 REMAINING THREE AML. 1433 01:04:04,715 --> 01:04:06,384 TIME OF DIAGNOSIS RANGED FROM 1434 01:04:06,384 --> 01:04:08,919 FOUR MONTHS TO 5 1/2 YEARS POST 1435 01:04:08,919 --> 01:04:09,353 TRANSPLANT. 1436 01:04:09,353 --> 01:04:10,921 AND WHEN WE GOT BONE MARROWS AT 1437 01:04:10,921 --> 01:04:14,125 THE TIME OF THE DIAGNOSIS, 1438 01:04:14,125 --> 01:04:15,993 THE -- WERE COMPLEX IN FOUR OF 1439 01:04:15,993 --> 01:04:18,062 THE PATIENTS, THE FIFTH PATIENT 1440 01:04:18,062 --> 01:04:20,364 HAD A SUDDEN ACUTE LESION. 1441 01:04:20,364 --> 01:04:22,166 FOUR OF THE PATIENTS COMPLETELY 1442 01:04:22,166 --> 01:04:24,001 REJECTED THEIR GRAFT. 1443 01:04:24,001 --> 01:04:27,805 THE FIFTH PATIENT HAD IMPENDING 1444 01:04:27,805 --> 01:04:29,707 FAILURE AND -- OF 18% AT THE 1445 01:04:29,707 --> 01:04:31,609 TIME OF AML DIAGNOSIS. 1446 01:04:31,609 --> 01:04:33,010 AND ALL OF THE PATIENTS ARE 1447 01:04:33,010 --> 01:04:36,213 DECEASED. 1448 01:04:36,213 --> 01:04:38,149 SO THIS TABLE SHOWS THE 1449 01:04:38,149 --> 01:04:42,353 INCIDENCE OF MDS OR AML ACROSS 1450 01:04:42,353 --> 01:04:43,988 PROTOCOL OR ACROSS STUDIES. 1451 01:04:43,988 --> 01:04:46,557 THESE FIRST THREE COLUMBUS SHOW 1452 01:04:46,557 --> 01:04:52,430 PATIENTS UNDERGOING HLA -- 1453 01:04:52,430 --> 01:04:56,400 EITHER AT THE NIH OR CHICAGO 1454 01:04:56,400 --> 01:04:58,235 OR -- YOU SEE THAT AT RISK TIME 1455 01:04:58,235 --> 01:05:02,506 RANGED FROM 87.5 TO 518 PERSON 1456 01:05:02,506 --> 01:05:04,575 YEARS WITH THE INCIDENCE OF MDS 1457 01:05:04,575 --> 01:05:08,679 OR AML RANGING FROM ZERO TO 0.8 1458 01:05:08,679 --> 01:05:09,213 PER 100 PERSON YEARS. 1459 01:05:09,213 --> 01:05:13,184 WITH THE STUDY THAT INCLUDED 1460 01:05:13,184 --> 01:05:14,785 PRECONDITIONING, AT RISK TIME IS 1461 01:05:14,785 --> 01:05:17,021 96 YEARS WITH THE INCIDENCE OF 1462 01:05:17,021 --> 01:05:24,628 MKM DS OR AML -- THIS FIRST COLN 1463 01:05:24,628 --> 01:05:28,666 SHOWS THE STUDY WHICH INCLUDES A 1464 01:05:28,666 --> 01:05:32,336 ESCALATION OF PT5, AS RISK TIME 1465 01:05:32,336 --> 01:05:35,840 WAS -- WITH INCIDENCE BEING 1466 01:05:35,840 --> 01:05:36,507 1.7 PER 100 PERSON YEARS. 1467 01:05:36,507 --> 01:05:38,943 THE STUDY THAT WE HAVE THAT 1468 01:05:38,943 --> 01:05:47,017 INCLUDES PENTOSTATIN/AM TEUZ 1469 01:05:47,017 --> 01:05:49,120 MAN -- WITH ALL FIVE OF THESE 1470 01:05:49,120 --> 01:05:50,421 STUDIES, THE THERAPEUTIC GOAL IS 1471 01:05:50,421 --> 01:05:53,424 MIXED DONOR AND RECIPIENT 1472 01:05:53,424 --> 01:05:53,724 CHIMERISM. 1473 01:05:53,724 --> 01:05:56,093 I'D LIKE TO THANK BLUEBIRD BIO, 1474 01:05:56,093 --> 01:05:57,728 THEY PROVIDED DATA ON THE NUMBER 1475 01:05:57,728 --> 01:05:59,029 OF PATIENTS AND PERSON YEARS AND 1476 01:05:59,029 --> 01:06:02,700 YOU CAN SEE THAT THEIR AT RISK 1477 01:06:02,700 --> 01:06:04,235 TIME WAS 112 PERSON YEARS AND 1478 01:06:04,235 --> 01:06:07,872 THE INCIDENCE OF AML WAS 1.79 1479 01:06:07,872 --> 01:06:09,006 PER 100 PERSON YEARS, WHICH IS 1480 01:06:09,006 --> 01:06:11,075 SIMILAR TO WHAT WE SEE IN OUR 1481 01:06:11,075 --> 01:06:14,245 STUDIES AT THE NIH. 1482 01:06:14,245 --> 01:06:21,018 BOTH WERE GROUP A PATIENTS AN 1483 01:06:21,018 --> 01:06:24,455 AND -- THIS COMPARES TO TWO 1484 01:06:24,455 --> 01:06:25,890 LARGER STUDIES WHICH IS MOSTLY 1485 01:06:25,890 --> 01:06:26,891 PEDIATRIC PATIENTS AND WHERE 1486 01:06:26,891 --> 01:06:29,560 BOTH OF THE PATIENTS UNDERWENT 1487 01:06:29,560 --> 01:06:30,528 MYELOBLAITIVE TRANSPLANT. 1488 01:06:30,528 --> 01:06:33,497 AT RISK TIME AGES FROM OVER 1600 1489 01:06:33,497 --> 01:06:34,832 TO 1800 PERSON YEARS, AND NONE 1490 01:06:34,832 --> 01:06:36,534 OF THE PATIENTS WERE REPORTED TO 1491 01:06:36,534 --> 01:06:38,702 DEVELOP MBS OR AML. 1492 01:06:38,702 --> 01:06:40,838 THE THERAPEUTIC GOAL OF THESE 1493 01:06:40,838 --> 01:06:43,674 STUDIES IS FULL DONOR CHIMERISM. 1494 01:06:43,674 --> 01:06:46,076 SO THE INCIDENCE IS HIGHEST IN 1495 01:06:46,076 --> 01:06:51,482 ADULTS WITH GRAFT FAILURE -- 1496 01:06:51,482 --> 01:06:52,383 GENE THERAPY FOR SICKLE CELL 1497 01:06:52,383 --> 01:06:52,816 DISEASE. 1498 01:06:52,816 --> 01:06:54,985 I MENTIONED EARLIER THAT THE 1499 01:06:54,985 --> 01:06:56,820 ABSOLUTE RISK OF AML IS LOW IN 1500 01:06:56,820 --> 01:06:58,122 PATIENTS WITH SICKLE CELL WHO 1501 01:06:58,122 --> 01:07:01,091 DON'T UNDERGO CURATIVE THAIMPLET 1502 01:07:01,091 --> 01:07:04,128 WE REPORTED THREE OF OUR 1503 01:07:04,128 --> 01:07:08,365 PATIENTS DEVELOPING AML -- SO 1504 01:07:08,365 --> 01:07:11,735 THE RATE OF AML IS APPROXIMATELY 1505 01:07:11,735 --> 01:07:13,204 89 TIMES HIGHER IN ADULTS 1506 01:07:13,204 --> 01:07:14,004 FOLLOWING TRANSPLANT FOR SICKLE 1507 01:07:14,004 --> 01:07:15,940 CELL DISEASE, RESULTING IN GRAFT 1508 01:07:15,940 --> 01:07:17,141 FAILURE COMPARED TO THOSE WITH 1509 01:07:17,141 --> 01:07:18,742 SICKLE CELL DISEASE WHO DO NOT 1510 01:07:18,742 --> 01:07:21,712 RECEIVE CURATIVE THERAPY. 1511 01:07:21,712 --> 01:07:23,681 WHY IS THE INCIDENCE OF 1512 01:07:23,681 --> 01:07:25,216 AGGRESSIVE MYELOID MALIGNANCIES 1513 01:07:25,216 --> 01:07:27,017 HIGHER IN ADULTS AFTER GRAFT 1514 01:07:27,017 --> 01:07:27,952 FAILURE AND GENE THERAPY FOR 1515 01:07:27,952 --> 01:07:30,854 SICKLE CELL DISEASE? 1516 01:07:30,854 --> 01:07:31,722 THERE'S MULTIPLE POSSIBLE 1517 01:07:31,722 --> 01:07:33,490 REASONS THAT NEED FURTHER 1518 01:07:33,490 --> 01:07:33,924 EX-PARTICULARRATION. 1519 01:07:33,924 --> 01:07:35,392 I SAID EARLIER THAT PATIENTS 1520 01:07:35,392 --> 01:07:36,927 WITH SICKLE CELL DISEASE WHO ARE 1521 01:07:36,927 --> 01:07:38,562 OLDER AND WHO HAD MORE SEVERE 1522 01:07:38,562 --> 01:07:41,031 DISEASE WERE MORE LIKELY TO 1523 01:07:41,031 --> 01:07:41,532 DEVELOP LEUKEMIA. 1524 01:07:41,532 --> 01:07:43,901 AT THE NIH, WE TRANSPLANT OLDER 1525 01:07:43,901 --> 01:07:45,202 PATIENTS THAT ARE TYPICAL AND 1526 01:07:45,202 --> 01:07:46,837 PATIENTS WITH MORE SEVERE 1527 01:07:46,837 --> 01:07:54,345 DISEASE THAT ARE -- COMPARED TO 1528 01:07:54,345 --> 01:07:56,947 THE PEDIATRIC POPULATION, WE USE 1529 01:07:56,947 --> 01:07:59,250 TOTAL BODY RADIATION VERSUS 1530 01:07:59,250 --> 01:08:00,851 CHEMOTHERAPY-BASED CONDITIONING, 1531 01:08:00,851 --> 01:08:02,386 AND IN THE SETTING OF GRAFT 1532 01:08:02,386 --> 01:08:04,655 FAILURE AND ALSO WITH GENE 1533 01:08:04,655 --> 01:08:06,857 THERAPY, THERE'S A REQUIREMENT 1534 01:08:06,857 --> 01:08:09,326 OF REGENERATIVE HEMATOPOEISIS. 1535 01:08:09,326 --> 01:08:11,829 SO BEFORE GOING ON, I WANT TO 1536 01:08:11,829 --> 01:08:15,032 TALK ABOUT THE HEALTHY 1537 01:08:15,032 --> 01:08:15,899 POPULATION AND IMPORTANT TO NOTE 1538 01:08:15,899 --> 01:08:21,272 THAT IN THIS STUDY, CLONAL 1539 01:08:21,272 --> 01:08:23,807 HEMATOPOEISIS -- FREQUENCY OF 1540 01:08:23,807 --> 01:08:26,810 AROUND -- MUTATIONS THAT HAD A 1541 01:08:26,810 --> 01:08:30,080 GRANULAR FREQUENCY OF 3-POINT -- 1542 01:08:30,080 --> 01:08:30,748 PERCENT OR HIGHER. 1543 01:08:30,748 --> 01:08:33,350 YOU CAN SEE THAT THE PREVALENCE 1544 01:08:33,350 --> 01:08:35,819 OF CLONAL HEMATOPOEISIS IS RARE 1545 01:08:35,819 --> 01:08:37,254 IN PEOPLE LESS THAN 40 YEARS OF 1546 01:08:37,254 --> 01:08:39,456 AGE AND IT INCREASES 10% AND 1547 01:08:39,456 --> 01:08:42,126 HIGHER IN THOSE WHO WERE 7 YEARS 1548 01:08:42,126 --> 01:08:43,794 OF AGE AND OLDER. 1549 01:08:43,794 --> 01:08:47,298 ALSO IN THE HEALTHY POPULATION, 1550 01:08:47,298 --> 01:08:51,602 THE GENES MOST COMMONLY IMPACTED 1551 01:08:51,602 --> 01:09:02,079 ARE DNMT3A, ASXL1 AND TET2. 1552 01:09:04,848 --> 01:09:06,317 SO I'VE SAID WE'VE HAD FIVE 1553 01:09:06,317 --> 01:09:08,085 PATIENTS DEVELOP MDS OR AML 1554 01:09:08,085 --> 01:09:09,353 AFTER OUR TRANSPLANT, AND FOUR 1555 01:09:09,353 --> 01:09:10,421 OF THE PATIENTS HAD NEXT 1556 01:09:10,421 --> 01:09:11,422 GENERATION SEQUENCING DONE AT 1557 01:09:11,422 --> 01:09:14,024 THE TIME OF THE DIAGNOSIS, AND 1558 01:09:14,024 --> 01:09:18,295 ALL FOUR PATIENTS HAD PATHOGENIC 1559 01:09:18,295 --> 01:09:20,764 TP53 MUTATIONS. 1560 01:09:20,764 --> 01:09:22,099 SO WASN'TED TO KNOW IF THEY WERE 1561 01:09:22,099 --> 01:09:24,001 CAUSED BY RADIATION WE GAVE THE 1562 01:09:24,001 --> 01:09:24,935 PATIENTS OR AS A RESULT OF 1563 01:09:24,935 --> 01:09:27,604 SICKLE CELL DISEASE, THE STRESS 1564 01:09:27,604 --> 01:09:29,373 AND INFLAMMATION OR IF THEY WERE 1565 01:09:29,373 --> 01:09:30,908 ALREADY PRESENT AT BASELINE. 1566 01:09:30,908 --> 01:09:32,376 THIS FIGURE ACTUALLY SHOWS 1567 01:09:32,376 --> 01:09:32,776 MR. SMITH'S DATA. 1568 01:09:32,776 --> 01:09:34,878 YOU CAN SEE AT THE TIME OF HIS 1569 01:09:34,878 --> 01:09:37,247 MDS DIAGNOSIS, HE HAD A TP53 1570 01:09:37,247 --> 01:09:40,084 MUTATION WITH A -- FREQUENCY OF 1571 01:09:40,084 --> 01:09:40,417 24%. 1572 01:09:40,417 --> 01:09:42,519 WHEN WE WENT BACK TO HIS 1573 01:09:42,519 --> 01:09:43,954 BASELINE BLOOD SAMPLES THAT WE 1574 01:09:43,954 --> 01:09:45,155 COLLECTED BEFORE WE DID 1575 01:09:45,155 --> 01:09:46,590 ANYTHING, THAT SAME MUTATION WAS 1576 01:09:46,590 --> 01:09:49,026 PRESENT WITH THE GRANULAR 1577 01:09:49,026 --> 01:09:51,762 FREQUENCY MUCH LOWER AT 0.3%. 1578 01:09:51,762 --> 01:09:54,832 AFTER GRAFT REJECTION, THE 1579 01:09:54,832 --> 01:09:56,300 PATIENT SUBSEQUENTLY DEVELOPED 1580 01:09:56,300 --> 01:09:56,934 MDS. 1581 01:09:56,934 --> 01:09:58,369 IN THE SECOND FIGURE, YOU SEE 1582 01:09:58,369 --> 01:10:00,904 OUR SECOND PATIENT WHO DEVELOPED 1583 01:10:00,904 --> 01:10:03,607 MDS AND HIS TP53 MUTATION WAS 1584 01:10:03,607 --> 01:10:05,576 DIFFERENT AND IT HAD A VERY LOW 1585 01:10:05,576 --> 01:10:06,710 FREQUENCY OF JUST ABOUT 3% AT 1586 01:10:06,710 --> 01:10:09,012 THE TIME OF HIS MDS DIAGNOSIS, 1587 01:10:09,012 --> 01:10:10,447 AND WHEN WE WENT BACK TO 1588 01:10:10,447 --> 01:10:11,849 BASELINE SAMPLES, THAT SAME 1589 01:10:11,849 --> 01:10:13,817 MUTATION WAS PRESENT, MUCH 1590 01:10:13,817 --> 01:10:15,119 LOWER, VERY LOW FREQUENCY, AS 1591 01:10:15,119 --> 01:10:18,489 WELL AS 0.03%. 1592 01:10:18,489 --> 01:10:21,892 GRAFFITI AFTER GRAFT REJECTION 1593 01:10:21,892 --> 01:10:23,994 IT EXPANDED AND THE PATIENT 1594 01:10:23,994 --> 01:10:24,661 DEVELOPED MDS. 1595 01:10:24,661 --> 01:10:28,165 SO THESE ARE THE RESULTS I JUST 1596 01:10:28,165 --> 01:10:30,734 TOLD YOU ABOUT THE OTHER TWO 1597 01:10:30,734 --> 01:10:31,635 PATIENTS. 1598 01:10:31,635 --> 01:10:34,872 A THIRD PATIENT HAD A TP53 1599 01:10:34,872 --> 01:10:37,374 MUTATION WITH A FREQUENCY OF 58% 1600 01:10:37,374 --> 01:10:39,676 AND AGAIN WE WENT BACK TO THE 1601 01:10:39,676 --> 01:10:42,513 SAME BASELINE SAMPLE, THE SAME 1602 01:10:42,513 --> 01:10:44,381 MUTATION WAS PRESENT, AND THE 1603 01:10:44,381 --> 01:10:46,650 FOURTH PATIENT HAD A TP53 1604 01:10:46,650 --> 01:10:51,321 MUTATION WITH A DIF OF 79% OF 1605 01:10:51,321 --> 01:10:52,823 AML DIAGNOSIS AND WHEN WE WENT 1606 01:10:52,823 --> 01:10:54,324 BACK TO THE BASELINE EXAMPLE, WE 1607 01:10:54,324 --> 01:11:02,499 WERE NOT ABLE TO DETECT, -- 1608 01:11:02,499 --> 01:11:04,368 0.06%, AND INCREASE ONE YEAR 1609 01:11:04,368 --> 01:11:07,638 LATER TO 0.34% AND SIX MONTHS 1610 01:11:07,638 --> 01:11:10,974 LATER IT INCREASED TO THE 79% 1611 01:11:10,974 --> 01:11:12,609 AND SHE DEVELOPED AML. 1612 01:11:12,609 --> 01:11:14,745 SO AT BASELINE, EITHER THE 1613 01:11:14,745 --> 01:11:16,013 MUTATION WAS AT SUCH A LEVEL WE 1614 01:11:16,013 --> 01:11:17,481 WEREN'T ABLE TO DETECT IT OR IT 1615 01:11:17,481 --> 01:11:19,049 WASN'T PRESENT. 1616 01:11:19,049 --> 01:11:22,052 SO WHAT ABOUT OUR PATIENTS WHO 1617 01:11:22,052 --> 01:11:24,121 ARE NOT DEVELOPING LEUKEMIA? 1618 01:11:24,121 --> 01:11:26,657 WE WANTED TO LOOK AT THE TP53 1619 01:11:26,657 --> 01:11:27,524 MUTATION FIRST IN A SET OF 1620 01:11:27,524 --> 01:11:29,860 PATIENTS WHO HAD BROAD FAILURE. 1621 01:11:29,860 --> 01:11:31,528 SO ALL OF THE CASES EXCEPT ONE 1622 01:11:31,528 --> 01:11:32,629 HAD HOMOZYGOUS SICKLE CELL 1623 01:11:32,629 --> 01:11:32,863 DISEASE. 1624 01:11:32,863 --> 01:11:35,499 ALL OF THEM HAPLOIDENTICAL 1625 01:11:35,499 --> 01:11:35,799 TRANSPLANT. 1626 01:11:35,799 --> 01:11:37,701 THE AGE AT THE TIME OF 1627 01:11:37,701 --> 01:11:39,303 TRANSPLANT RANGED FROM 20 TO 506 1628 01:11:39,303 --> 01:11:39,570 YEARS. 1629 01:11:39,570 --> 01:11:42,506 NONE OF THEM WERE FOUND TO HAVE 1630 01:11:42,506 --> 01:11:44,007 TP53 MUTATIONS BEFORE THE 1631 01:11:44,007 --> 01:11:44,341 TRANSPLANT. 1632 01:11:44,341 --> 01:11:45,542 THREE OF THE PATIENTS ARE 1633 01:11:45,542 --> 01:11:48,679 DECEASED AND FOLLOW-UP RANGED 1634 01:11:48,679 --> 01:11:53,250 FROM 40 1/2 TO 12 YEARS. 1635 01:11:53,250 --> 01:11:54,585 THE EIGHT PATIENTS REMAINING 1636 01:11:54,585 --> 01:11:56,753 GRAFTED, ALL HAD HOMOZYGOUS 1637 01:11:56,753 --> 01:11:59,156 SICKLE CELL DISEASE, HALF 1638 01:11:59,156 --> 01:12:00,224 UNDERWENT HAPLOIDENTICAL 1639 01:12:00,224 --> 01:12:01,425 TRANSPLANT, AGE RANGE FROM 10 TO 1640 01:12:01,425 --> 01:12:02,459 50 YEARS AT THE TIME OF 1641 01:12:02,459 --> 01:12:03,794 TRANSPLANT AND AGAIN, WE DID NOT 1642 01:12:03,794 --> 01:12:06,964 DETECT ANY TP53 MUTATIONS AT 1643 01:12:06,964 --> 01:12:08,565 BASELINE WITH FOLLOW-UP RANGING 1644 01:12:08,565 --> 01:12:10,200 FROM 4 YEARS TO 9 YEARS POST 1645 01:12:10,200 --> 01:12:13,036 TRANSPLANT. 1646 01:12:13,036 --> 01:12:16,540 WHAT ABOUT THE BASELINE 1647 01:12:16,540 --> 01:12:17,541 PREVALENCE IN PATIENTS WITH 1648 01:12:17,541 --> 01:12:18,775 SICKLE CELL DISEASE WHO HAVE NOT 1649 01:12:18,775 --> 01:12:20,544 RECEIVED CURATIVE THERAPY? 1650 01:12:20,544 --> 01:12:22,513 A VERY LOW FREQUENCY OF 10% IS 1651 01:12:22,513 --> 01:12:24,147 ASSOCIATED WITH A PREDISPOSITION 1652 01:12:24,147 --> 01:12:27,184 TO MYELOID MALIGNANCY 1653 01:12:27,184 --> 01:12:29,052 DEVELOPMENT, SO -- AND 1654 01:12:29,052 --> 01:12:31,989 COLLEAGUES ANALYZED WHOLE GENOME 1655 01:12:31,989 --> 01:12:35,225 SEQUENCING DATA AND 71,000 1656 01:12:35,225 --> 01:12:35,993 UNAFFECTED CONTROLS. 1657 01:12:35,993 --> 01:12:39,363 WE WERE ABLE TO RELIABLY DETECT 1658 01:12:39,363 --> 01:12:40,631 MOST MUTATIONS ABOVE 5% AND AT 1659 01:12:40,631 --> 01:12:41,965 THIS LEVEL, THEY WERE NOT ABLE 1660 01:12:41,965 --> 01:12:44,568 TO DETECT A DIFFERENCE IN THE 1661 01:12:44,568 --> 01:12:47,271 PREVALENCE OF CLONAL 1662 01:12:47,271 --> 01:12:50,107 HEMATOPOEISIS BETWEEN GROUPS. 1663 01:12:50,107 --> 01:12:52,609 -- AND COLLEAGUES ANALYZED 1664 01:12:52,609 --> 01:12:55,379 SEQUENCING DATA IN 1400 1665 01:12:55,379 --> 01:12:56,079 INDIVIDUALS WITH SICKLE CELL 1666 01:12:56,079 --> 01:12:57,614 DISEASE AND JUST UNDER 7,000 1667 01:12:57,614 --> 01:12:58,382 UNAFFECTED CONTROLS. 1668 01:12:58,382 --> 01:13:01,218 THEY WERE ABLE TO DETECT THE 1669 01:13:01,218 --> 01:13:04,021 AMOUNT OF -- AS WELL AS 2 1/2%. 1670 01:13:04,021 --> 01:13:05,455 THE MEAN AGE OF THE PATIENTS 1671 01:13:05,455 --> 01:13:07,090 WITH SICKLE CELL DISEASE WAS 1672 01:13:07,090 --> 01:13:09,626 YOUNGER, AT 24 YEARS VERSUS 50 1673 01:13:09,626 --> 01:13:11,862 YEARS, AND DESPITE THEM BEING 1674 01:13:11,862 --> 01:13:16,233 YUN GER, THEY FOUND 15 -- 1675 01:13:16,233 --> 01:13:17,668 MUTATIONS AND -- WITH SICKLE 1676 01:13:17,668 --> 01:13:20,003 CELL DISEASE WITH YOUNGEST BEING 1677 01:13:20,003 --> 01:13:23,240 17 YEARS AND 199 MUTATIONS AND 1678 01:13:23,240 --> 01:13:24,942 189 CONTROLS WITH THE YOUNGEST 1679 01:13:24,942 --> 01:13:26,310 BEING 34 YEARS. 1680 01:13:26,310 --> 01:13:27,844 THEY ALSO FOUND TWO PATIENTS 1681 01:13:27,844 --> 01:13:29,146 BETWEEN 23 AND 36 YEARS OF AGE 1682 01:13:29,146 --> 01:13:32,816 WHO HAD TP53 MUTATIONS WITH A 1683 01:13:32,816 --> 01:13:34,918 VAF RANGING FROM 7% TO 26%, AND 1684 01:13:34,918 --> 01:13:38,288 THIS IS DEFINITELY HIGHER 1685 01:13:38,288 --> 01:13:39,456 FREQUENCY THAN WE WOULD HAVE 1686 01:13:39,456 --> 01:13:39,790 EXPECTED. 1687 01:13:39,790 --> 01:13:41,525 SICKLE CELL DISEASE STATUS WAS 1688 01:13:41,525 --> 01:13:42,726 INDEPENDENTLY ASSOCIATED WITH 1689 01:13:42,726 --> 01:13:44,161 THE INCREASE IN CH, AND AT THE 1690 01:13:44,161 --> 01:13:47,397 AGE OF 50 YEARS, THE PROBABILITY 1691 01:13:47,397 --> 01:13:50,133 TO DETECT THE CH MUTATION WAS 7% 1692 01:13:50,133 --> 01:13:53,036 IN SICKLE CELL DISEASE AND 0.7% 1693 01:13:53,036 --> 01:13:57,007 IN CONTROLS. 1694 01:13:57,007 --> 01:13:57,741 IT'S ALSO IMPORTANT TO REMEMBER 1695 01:13:57,741 --> 01:14:01,044 THAT THE TRUE PREVALENCE OF 1696 01:14:01,044 --> 01:14:03,513 ULTRA DEEP HEMATOPOEISIS IN 1697 01:14:03,513 --> 01:14:04,114 PATIENTS WITH SICKLE CELL 1698 01:14:04,114 --> 01:14:06,249 DISEASE MAY BE HIGHER AND 1699 01:14:06,249 --> 01:14:07,985 CLINICALLY RELEVANT IN THE 1700 01:14:07,985 --> 01:14:10,354 SETTING OF -- I'M WORKING ON A 1701 01:14:10,354 --> 01:14:11,221 MULTICENTER COLLABORATION 1702 01:14:11,221 --> 01:14:13,957 THROUGH A U01 AWARD WITH 1703 01:14:13,957 --> 01:14:16,693 INVESTIGATORS AT HOPKINS AND 1704 01:14:16,693 --> 01:14:19,796 VANDERBILT AND OTHER SITES 1705 01:14:19,796 --> 01:14:20,797 CALLED COALESCE, AND WE'RE 1706 01:14:20,797 --> 01:14:22,432 LOOKING AT THE LONG TERM HEALTH 1707 01:14:22,432 --> 01:14:24,501 EFFECTS OF CURATIVE THERAPIES IN 1708 01:14:24,501 --> 01:14:26,003 PATIENTS WITH SICKLE CELL 1709 01:14:26,003 --> 01:14:27,337 DISEASE AND WE HIGH POTASSIUM 1710 01:14:27,337 --> 01:14:30,507 SEISE THAT AFTER GRAFT FAILURE 1711 01:14:30,507 --> 01:14:34,111 OR GENE THERAPY, THE STRESS OF 1712 01:14:34,111 --> 01:14:37,147 SWITCHING FROM HOMEOSTATIC TO 1713 01:14:37,147 --> 01:14:39,082 REGENERATIVE HEMATOPOEISIS BY 1714 01:14:39,082 --> 01:14:41,284 AUTOLOGOUS CELLS DRIVES CLONAL 1715 01:14:41,284 --> 01:14:43,553 EXPANSION AND TRANSFORMATION OF 1716 01:14:43,553 --> 01:14:44,655 PRE-EXISTING PRELIG NANT CLONES 1717 01:14:44,655 --> 01:14:48,025 EVENTUALLY RESULTING IN AML/MDS. 1718 01:14:48,025 --> 01:14:49,760 HOW TO POTENTIALLY MITIGATE THE 1719 01:14:49,760 --> 01:14:50,627 RISK. 1720 01:14:50,627 --> 01:14:56,199 SO THIS REPRESENTS -- AGING AND 1721 01:14:56,199 --> 01:14:57,267 COMORBIDITIES THEY MAY DEVELOP 1722 01:14:57,267 --> 01:14:59,336 THIS LOW LEVEL SOMATIC MUTATION 1723 01:14:59,336 --> 01:15:01,004 THAT IF THEY DON'T RECEIVE 1724 01:15:01,004 --> 01:15:02,072 CURATIVE THERAPY, THEY DON'T 1725 01:15:02,072 --> 01:15:03,373 EXPAND SO THAT MOST PATIENTS 1726 01:15:03,373 --> 01:15:04,474 WITH SICKLE CELL DISEASE DON'T 1727 01:15:04,474 --> 01:15:07,177 DEVELOP MYELOID MALIGNANCIES. 1728 01:15:07,177 --> 01:15:08,845 HOWEVER, IN THE SETTING OF GRAFT 1729 01:15:08,845 --> 01:15:11,748 REJECTION OR GENE THERAPY, A 1730 01:15:11,748 --> 01:15:12,949 PERIPHERAL STRESS INVOLVED LEADS 1731 01:15:12,949 --> 01:15:14,918 TO CLONAL EXPANSION AND FUTURE 1732 01:15:14,918 --> 01:15:16,219 MYELOID MALIGNANCY DEVELOPMENT. 1733 01:15:16,219 --> 01:15:18,055 I MENTIONED THAT BOTH OF THE 1734 01:15:18,055 --> 01:15:19,256 GENE THERAPY PATIENTS ARE IN 1735 01:15:19,256 --> 01:15:21,458 GROUP A, SO THE CELL DOSE THEY 1736 01:15:21,458 --> 01:15:22,726 RECEIVED WAS LOWER AND THEY 1737 01:15:22,726 --> 01:15:25,328 POTENTIALLY HAD TO UNDERGO 1738 01:15:25,328 --> 01:15:26,663 MORE -- STRESS, SO THAT'S A 1739 01:15:26,663 --> 01:15:29,466 POSSIBLE REASON WE'VE ONLY SEE 1740 01:15:29,466 --> 01:15:30,801 AML IN GROUP A PATIENTS TO DATE. 1741 01:15:30,801 --> 01:15:32,536 IF THERE WAS A GENETIC RISK 1742 01:15:32,536 --> 01:15:34,271 FACTOR WE'RE ABLE TO IDENTIFY 1743 01:15:34,271 --> 01:15:37,207 AND THE PATIENT HAD IT, WE COULD 1744 01:15:37,207 --> 01:15:40,177 RECOMMEND ALLOGENEIC -- THAT'S 1745 01:15:40,177 --> 01:15:41,678 BECAUSE MOST DONORS ARE YOUNG SO 1746 01:15:41,678 --> 01:15:44,147 THEY'RE NOT LIKELY TO HAVE 1747 01:15:44,147 --> 01:15:48,018 CLONAL HEMATOPEE HEMATOPOEISIS- 1748 01:15:48,018 --> 01:15:48,385 EXTREMELY RARE. 1749 01:15:48,385 --> 01:15:50,020 IF THEY DIDN'T HAVE A GENETIC 1750 01:15:50,020 --> 01:15:51,855 RISK FACTOR, WE COULD RECOMMEND 1751 01:15:51,855 --> 01:15:53,890 ANY KIND OF CURATIVE THERAPY 1752 01:15:53,890 --> 01:15:55,325 INCLUDING GENE THERAPY OND GENE 1753 01:15:55,325 --> 01:15:58,395 EDITING BUT UNFORTUNATELY 1754 01:15:58,395 --> 01:16:01,264 THERE'S NO -- THAT ACTUALLY 1755 01:16:01,264 --> 01:16:02,566 EXIST -- IN MY LAB OR OUTSIDE 1756 01:16:02,566 --> 01:16:03,300 THE NIH. 1757 01:16:03,300 --> 01:16:06,069 SO HOW DO WE COUNSEL PATIENTS 1758 01:16:06,069 --> 01:16:10,173 ABOUT THE BASELINE RISK -- I 1759 01:16:10,173 --> 01:16:12,275 WROTE THIS WILL MANUSCRIPT AND 1760 01:16:12,275 --> 01:16:14,311 THIS REPRESENTS OUR OPINION. 1761 01:16:14,311 --> 01:16:19,483 THE PRESENCE OF CH MAY GUIDE THE 1762 01:16:19,483 --> 01:16:20,984 THERAPEUTIC DECISIONS IN 1763 01:16:20,984 --> 01:16:21,518 PATIENTS WITH SICKLE CELL 1764 01:16:21,518 --> 01:16:21,852 DISEASE. 1765 01:16:21,852 --> 01:16:24,221 INDIVIDUALS WITH HIGH RISK SUCH 1766 01:16:24,221 --> 01:16:26,857 AS TP53 MUTATIONS SHOULD BE 1767 01:16:26,857 --> 01:16:27,991 OFFERED CURATIVE THERAPIES 1768 01:16:27,991 --> 01:16:30,227 INTENDED TO INDUCE FULL DONOR 1769 01:16:30,227 --> 01:16:32,162 CHIMERISM. 1770 01:16:32,162 --> 01:16:33,797 ALTERNATIVELY, PATIENTS WITHOUT 1771 01:16:33,797 --> 01:16:35,766 DETECTABLE CH OR HEREDITARY 1772 01:16:35,766 --> 01:16:37,267 CANCER PREDISPOSITION SYNDROMES 1773 01:16:37,267 --> 01:16:38,835 COULD BE OFFERED GENE 1774 01:16:38,835 --> 01:16:42,005 THERAPY/GENE EDITING. 1775 01:16:42,005 --> 01:16:43,707 NO RELIABLE BASELINE SCREENING 1776 01:16:43,707 --> 01:16:45,242 STUDIES ARE CURRENTLY AVAILABLE 1777 01:16:45,242 --> 01:16:47,410 TO ASSESS THE RISK OF MYELOID 1778 01:16:47,410 --> 01:16:49,146 MALIGNANCY DEVELOPMENT AFTER 1779 01:16:49,146 --> 01:16:50,347 CURATIVE THERAPY FOR SICKLE CELL 1780 01:16:50,347 --> 01:16:51,348 DISEASE. 1781 01:16:51,348 --> 01:16:56,253 SO IN SUMMARY, HLA MATCHED -- 1782 01:16:56,253 --> 01:16:57,621 OFFER CURATIVE OPTIONS FOR 1783 01:16:57,621 --> 01:17:00,724 CHILDREN AND ACUL ADULTS WITH SE 1784 01:17:00,724 --> 01:17:04,427 CELL DISEASE. 1785 01:17:04,427 --> 01:17:05,729 WHILE -- THE ABSOLUTE RISK IS 1786 01:17:05,729 --> 01:17:07,430 LOW. 1787 01:17:07,430 --> 01:17:09,065 THE INCIDENCE OF MYELOID 1788 01:17:09,065 --> 01:17:10,167 MALIGNANCY DEVELOPMENT IS HIGHER 1789 01:17:10,167 --> 01:17:11,668 THAN EXPECTED AFTER GRAFT 1790 01:17:11,668 --> 01:17:14,971 FAILURE AND GHEEN THE GENE THERR 1791 01:17:14,971 --> 01:17:15,906 SICKLE CELL DISEASE. 1792 01:17:15,906 --> 01:17:16,807 WHILE WE DON'T KNOW WHY OUR 1793 01:17:16,807 --> 01:17:19,376 PATIENTS HAVE A HIGHER RISK OF 1794 01:17:19,376 --> 01:17:21,244 MALIGNANT MYELOID MALIGNANCIES, 1795 01:17:21,244 --> 01:17:23,980 FUTURE PROTOCOLS HAVE SHIFTED 1796 01:17:23,980 --> 01:17:26,349 FROM -- TO FULL DONOR CHIMERISM. 1797 01:17:26,349 --> 01:17:27,784 NO RELIABLE BASELINE SCREENING 1798 01:17:27,784 --> 01:17:28,885 STUDIES ARE CURRENTLY AVAILABLE 1799 01:17:28,885 --> 01:17:31,788 TO ASSESS THE RISK OF MYELOID 1800 01:17:31,788 --> 01:17:32,889 MALIGNANCY DEVELOPMENT AFTER 1801 01:17:32,889 --> 01:17:34,090 CURATIVE THERAPY FOR SICKLE CELL 1802 01:17:34,090 --> 01:17:35,158 DISEASE. 1803 01:17:35,158 --> 01:17:37,160 THE RISK OF MYELOID MALIGNANCY 1804 01:17:37,160 --> 01:17:38,328 DEVELOPMENT AFTER CURATIVE 1805 01:17:38,328 --> 01:17:39,196 THERAPY FOR SICKLE CELL DISEASE 1806 01:17:39,196 --> 01:17:40,397 SHOULD BE CONSIDERED IN THE 1807 01:17:40,397 --> 01:17:42,165 CONTEXT OF THE RISK OF EARLY 1808 01:17:42,165 --> 01:17:43,900 MORTALITY IN ADULTS WITH SICKLE 1809 01:17:43,900 --> 01:17:45,836 CELL DISEASE. 1810 01:17:45,836 --> 01:17:47,370 AND THE LONG-TERM FOLLOW-UP IS 1811 01:17:47,370 --> 01:17:49,239 CRUCIAL TO EVALUATE THE 1812 01:17:49,239 --> 01:17:50,006 LONG-TERM HEALTH EFFECT OF 1813 01:17:50,006 --> 01:17:50,841 CURATIVE THERAPY IN PATIENTS 1814 01:17:50,841 --> 01:17:52,275 WITH SICKLE CELL DISEASE. 1815 01:17:52,275 --> 01:17:54,644 AND I WANT US TO END BY TALKING 1816 01:17:54,644 --> 01:17:58,148 ABOUT THE DIRECTION THE -- IS 1817 01:17:58,148 --> 01:17:59,349 GOING IN ORDER TO TRY TO 1818 01:17:59,349 --> 01:18:01,518 INCREASE THE CHANCE OF -- 1819 01:18:01,518 --> 01:18:02,085 CHIMERISM. 1820 01:18:02,085 --> 01:18:04,621 SO I'VE TOLD YOU WE'VE HAD TWO 1821 01:18:04,621 --> 01:18:06,223 DIFFERENT -- PROTOCOLS TO DATE. 1822 01:18:06,223 --> 01:18:07,858 THE Y AXIS SHOWS THE PROPORTION 1823 01:18:07,858 --> 01:18:09,159 OF PATIENTS WHO HAVE NOT 1824 01:18:09,159 --> 01:18:09,793 EXPERIENCED REJECTION, AND YOU 1825 01:18:09,793 --> 01:18:13,296 CAN SEE WITH THE SECOND PHOTO WE 1826 01:18:13,296 --> 01:18:16,566 ADDED -- WITH A SIGNIFICANTLY 1827 01:18:16,566 --> 01:18:18,735 HIGHER PROPORTION -- GRAFT 1828 01:18:18,735 --> 01:18:19,502 REJECTION SO THAT'S THE GOOD 1829 01:18:19,502 --> 01:18:20,136 NEWS. 1830 01:18:20,136 --> 01:18:23,440 THE BAD NEWS IS THAT TWO 1831 01:18:23,440 --> 01:18:28,979 PATIENTS DEVELOPED -- FIVE -- 1832 01:18:28,979 --> 01:18:29,846 PROLIFERATIVE DISEASE WITH ONE 1833 01:18:29,846 --> 01:18:32,782 OF THEM DYING, SO THE PROTOCOL 1834 01:18:32,782 --> 01:18:34,818 CLOSED TO A COOL IN NOVEMBER OF 1835 01:18:34,818 --> 01:18:36,486 2023. 1836 01:18:36,486 --> 01:18:38,788 SO WITH OUR FIRST HAPLOIDENTICAL 1837 01:18:38,788 --> 01:18:40,190 PROTOCOL, THERE WAS A HIGH RATE 1838 01:18:40,190 --> 01:18:42,392 OF GRAPH REJECTION AND 1839 01:18:42,392 --> 01:18:43,994 AGGRESSIVE MYELOID MALIGNANCY. 1840 01:18:43,994 --> 01:18:46,396 SO WE HYPOTHESIZED WE DIDN'T 1841 01:18:46,396 --> 01:18:49,032 GIVE THEM ENOUGH 1842 01:18:49,032 --> 01:18:49,900 IMMUNOSUPPRESSION. 1843 01:18:49,900 --> 01:18:51,001 WITH THE SECOND PROTOCOL, THERE 1844 01:18:51,001 --> 01:18:52,836 WAS A HIGH RATE OF VIRAL DISEASE 1845 01:18:52,836 --> 01:18:54,037 AND HYPERINFLAMMATORY 1846 01:18:54,037 --> 01:18:56,406 COMPLICATIONS, SO WE HAD 1847 01:18:56,406 --> 01:18:58,041 HYPOTHESIZED WE GAVE THEM TOO 1848 01:18:58,041 --> 01:18:58,541 MUCH IMMUNOSUPPRESSION. 1849 01:18:58,541 --> 01:19:01,144 SO WE NOW HYPOTHESIZE THAT A 1850 01:19:01,144 --> 01:19:05,782 MODERATE AMOUNT OF AMINO -- AND 1851 01:19:05,782 --> 01:19:07,117 INCREASED MILE LOW SUPPRESSION 1852 01:19:07,117 --> 01:19:10,086 WILL MAINTAIN A LOW RATE OF 1853 01:19:10,086 --> 01:19:16,059 GRAFT REJECTION WHILE AVOIDING 1854 01:19:16,059 --> 01:19:16,826 UNACCEPTABLE TOXICITIES. 1855 01:19:16,826 --> 01:19:19,429 SO THIS FIGURE HERE SHOWS THE 1856 01:19:19,429 --> 01:19:20,931 ANTIGEN ENGAGING THE T-CELL 1857 01:19:20,931 --> 01:19:21,364 RECEPTOR. 1858 01:19:21,364 --> 01:19:22,799 THIS IS KNOWN AS SIGNAL ONE. 1859 01:19:22,799 --> 01:19:24,234 AND IN THE SETTING OF 1860 01:19:24,234 --> 01:19:25,368 CO-STIMULATION OR SIGNAL TWO, 1861 01:19:25,368 --> 01:19:29,339 THERE'S A NORMAL IMMUNE 1862 01:19:29,339 --> 01:19:30,740 RESPONSE. 1863 01:19:30,740 --> 01:19:33,710 THE -- ALLOWS -- SO THE T-CELL 1864 01:19:33,710 --> 01:19:36,947 ENTERS AN ANERGIC STATE AND 1865 01:19:36,947 --> 01:19:39,683 WE'RE -- SO WITH THIS NEW 1866 01:19:39,683 --> 01:19:43,420 PROTOCOL, DETAILING FOR IG, AND 1867 01:19:43,420 --> 01:19:44,921 IT ALSO -- TO OCCUR BUT IT 1868 01:19:44,921 --> 01:19:49,926 BLOCKS SIGNAL TWO, SO -- 1869 01:19:49,926 --> 01:19:50,527 IMMUNOLOGIC TOLERANCE. 1870 01:19:50,527 --> 01:19:53,663 SO THE PROTOCOL -- MODERATE 1871 01:19:53,663 --> 01:19:55,265 AMOUNT OF IMMUNOSUPPRESSION. 1872 01:19:55,265 --> 01:19:57,467 THE INCREASING AMOUNT OF 1873 01:19:57,467 --> 01:20:00,403 MYELOSUPPRESSION, WE'VE INCLUDED 1874 01:20:00,403 --> 01:20:03,106 AN UNCONJUGATED ANTIBODY THAT 1875 01:20:03,106 --> 01:20:05,742 FUNCTIONALLY BLOCKS INTERACTION 1876 01:20:05,742 --> 01:20:07,711 WITH STEM CELL FACTOR, LEADING 1877 01:20:07,711 --> 01:20:08,678 TO STEM CELL DEATH. 1878 01:20:08,678 --> 01:20:10,847 SO THIS IS OUR NEW PROTOCOL THAT 1879 01:20:10,847 --> 01:20:12,382 JUST OPENED THIS YEAR. 1880 01:20:12,382 --> 01:20:14,451 THIS IS THE FIRST COHORT AND 1881 01:20:14,451 --> 01:20:17,687 THIS PROTOCOL IS TARGETING 1882 01:20:17,687 --> 01:20:18,922 PATIENTS WHO HAVE HEART, LUNG 1883 01:20:18,922 --> 01:20:21,024 AND KIDNEY IMPAIRMENT, INCLUDING 1884 01:20:21,024 --> 01:20:22,459 PATIENTS ON DIALYSIS, WHO ARE 1885 01:20:22,459 --> 01:20:25,128 NOT ABLE TO UNDERGO TRANSPLANT 1886 01:20:25,128 --> 01:20:26,329 OUTSIDE THE NIH. 1887 01:20:26,329 --> 01:20:28,298 SO WITH THE FIRST COHORT, 1888 01:20:28,298 --> 01:20:33,837 PATIENTS RECEIVED BRIQU LI. 1889 01:20:33,837 --> 01:20:42,645 LIMAB, ALEMTUZUMAB, AS WELL AS . 1890 01:20:42,645 --> 01:20:45,615 IF TOO MANY PATIENTS EXPERIENCE 1891 01:20:45,615 --> 01:20:46,583 REJECTION -- WHERE EVERYTHING 1892 01:20:46,583 --> 01:20:47,984 REMAINS THE SAME EXCEPT WE ADDED 1893 01:20:47,984 --> 01:20:50,520 A SECOND DOSE OF PT5. 1894 01:20:50,520 --> 01:20:52,022 WE TRANSPLANTED TWO PATIENTS AND 1895 01:20:52,022 --> 01:20:53,857 TO DATE, BOTH OF THEM ARE FREE 1896 01:20:53,857 --> 01:20:55,725 OF SICKLE CELL DISEASE. 1897 01:20:55,725 --> 01:20:57,427 SO I'D LIKE TO THANK THE 1898 01:20:57,427 --> 01:20:59,562 PATIENTS AND THEIR FAMILIES, I'D 1899 01:20:59,562 --> 01:21:00,930 LIKE TO THANK ALL MY LAB 1900 01:21:00,930 --> 01:21:07,604 MEMBERS, ESPECIALLY EMILY, THE 1901 01:21:07,604 --> 01:21:13,643 TISDALE LAB, THE HOURIGAN LAB 1902 01:21:13,643 --> 01:21:17,547 DID ALL THE TP53 SEQUENCING. 1903 01:21:17,547 --> 01:21:19,516 I'D LIKE TO THANK THE PROTOCOL 1904 01:21:19,516 --> 01:21:28,658 SUPPORT TEAM, THE ALESCE, NHLBI 1905 01:21:28,658 --> 01:21:30,326 FOR BEING AN ADVOCATE FOR ME AND 1906 01:21:30,326 --> 01:21:33,496 FOR PROVIDING FUNDING NOW THAT 1907 01:21:33,496 --> 01:21:36,499 WE'RE DOING SOME WORK ON GENETIC 1908 01:21:36,499 --> 01:21:43,006 RISK FACTORS, AND THE CLINICAL 1909 01:21:43,006 --> 01:21:43,940 STAFF OF THE CRC. 1910 01:21:43,940 --> 01:21:45,108 SO THANK YOU, I'M HAPPY TO TAKE 1911 01:21:45,108 --> 01:21:45,708 ANY QUESTIONS. 1912 01:21:45,708 --> 01:21:55,885 [APPLAUSE] 1913 01:21:58,755 --> 01:22:00,323 >> COURTNEY, THAT WAS BRILLIANT. 1914 01:22:00,323 --> 01:22:01,624 SO MANY QUESTIONS. 1915 01:22:01,624 --> 01:22:02,559 BUT JUST ONE. 1916 01:22:02,559 --> 01:22:05,161 AS YOU KNOW, THE CLINICAL 1917 01:22:05,161 --> 01:22:06,863 EXPRESSION AND THE GENETIC 1918 01:22:06,863 --> 01:22:08,932 ARCHITECTURE OF SICKLE CELL 1919 01:22:08,932 --> 01:22:10,767 DISEASE VARIES ACROSS SO MANY 1920 01:22:10,767 --> 01:22:14,504 DIFFERENT POPULATIONS. 1921 01:22:14,504 --> 01:22:17,240 THE QUESTION I HAVE WITH THE CH 1922 01:22:17,240 --> 01:22:19,742 MUTATION, THE PREVALENCE YOU 1923 01:22:19,742 --> 01:22:21,478 SHOWED, WHAT SICKLE CELL 1924 01:22:21,478 --> 01:22:23,480 POPULATIONS WERE STUDIED? 1925 01:22:23,480 --> 01:22:27,317 >> SO WE TRANSPLANT PATIENTS 1926 01:22:27,317 --> 01:22:28,651 FROM ALL OVER THE WORLD HERE, SO 1927 01:22:28,651 --> 01:22:30,653 WE HAVE TRANSPLANTED PATIENTS 1928 01:22:30,653 --> 01:22:33,256 FROM DIFFERENT COUNTRIES IN 1929 01:22:33,256 --> 01:22:35,024 AFRICA, SOME FROM THE CARIBBEAN, 1930 01:22:35,024 --> 01:22:36,226 FROM SOUTH AFRICA, FROM ALL 1931 01:22:36,226 --> 01:22:36,526 OVER. 1932 01:22:36,526 --> 01:22:37,961 THAT'S THE THING I REALLY LOVE 1933 01:22:37,961 --> 01:22:39,295 ABOUT NIH IS WE CAN TRANSPLANT 1934 01:22:39,295 --> 01:22:40,029 ANYBODY WE WANT. 1935 01:22:40,029 --> 01:22:41,464 SO OUR POPULATION IS VERY 1936 01:22:41,464 --> 01:22:42,198 DIVERSE. 1937 01:22:42,198 --> 01:22:43,166 IT'S MOSTLY AFRICAN AMERICANS 1938 01:22:43,166 --> 01:22:46,269 BUT THERE ARE PEOPLE FROM -- OF 1939 01:22:46,269 --> 01:22:46,803 THE COUNTRY. 1940 01:22:46,803 --> 01:22:49,506 WHEN YOU LOOK AT THE LARGER 1941 01:22:49,506 --> 01:22:52,909 STUDIES, IT'S ALL AMERICAN-BASED 1942 01:22:52,909 --> 01:22:53,776 STUDIES, MOSTLY AMERICANS. 1943 01:22:53,776 --> 01:22:58,248 >> RIGHT, SO OF THOSE PATIENTS S 1944 01:22:58,248 --> 01:23:00,450 WERE RESIDENTS IN THE UNITED 1945 01:23:00,450 --> 01:23:00,683 STATES? 1946 01:23:00,683 --> 01:23:02,185 >> IN THE LARGER STUDY BUT NOT 1947 01:23:02,185 --> 01:23:02,652 OUR STUDY. 1948 01:23:02,652 --> 01:23:04,087 >> OKAY, WE CAN TALK MORE ABOUT 1949 01:23:04,087 --> 01:23:06,556 THAT. 1950 01:23:06,556 --> 01:23:08,291 >> FIRST CONGRATULATIONS ON A 1951 01:23:08,291 --> 01:23:10,059 WONDERFUL TALK AND A GREAT BODY 1952 01:23:10,059 --> 01:23:11,995 OF WORK AND EXCITING TO SEE 1953 01:23:11,995 --> 01:23:12,562 WHERE THIS LEADS. 1954 01:23:12,562 --> 01:23:13,429 >> THANK YOU. 1955 01:23:13,429 --> 01:23:15,165 >> I HAD A QUESTION ON ONE OF 1956 01:23:15,165 --> 01:23:20,503 YOUR EARLIER SLIDES. 1957 01:23:20,503 --> 01:23:24,707 YOU MENTIONED THE POPULATION OF 1958 01:23:24,707 --> 01:23:26,409 SICKLE CELL PATIENTS, ONLY 60% 1959 01:23:26,409 --> 01:23:30,780 WERE ON HYDROXYCHLOROQUINE -- 1960 01:23:30,780 --> 01:23:31,514 HYDROXYUREA. 1961 01:23:31,514 --> 01:23:32,081 SORRY. 1962 01:23:32,081 --> 01:23:33,283 I'M WONDERING, THIS IS A LITTLE 1963 01:23:33,283 --> 01:23:35,051 OFF TARGET FROM WHERE YOUR WORK 1964 01:23:35,051 --> 01:23:36,753 IS, BUT HOW DO WE GET MORE OF 1965 01:23:36,753 --> 01:23:38,087 OUR PATIENTS WITH SICKLE CELL TO 1966 01:23:38,087 --> 01:23:40,156 GET WHAT THE STANDARD OF CARE 1967 01:23:40,156 --> 01:23:43,092 THERAPY SHOULD BE AT THE 1968 01:23:43,092 --> 01:23:43,326 PRESENT? 1969 01:23:43,326 --> 01:23:44,861 >> THAT'S A GREAT QUESTION, AND 1970 01:23:44,861 --> 01:23:47,463 I THINK THERE'S SOME FEAR 1971 01:23:47,463 --> 01:23:49,899 ASSOCIATED WITH HYDROXYUREA IN 1972 01:23:49,899 --> 01:23:51,267 PATIENTS AND EVEN IN PHYSICIANS 1973 01:23:51,267 --> 01:23:54,037 WHERE THERE IS A CONCERN, SO I 1974 01:23:54,037 --> 01:23:58,341 ALWAYS WANT TO MAKE THE POINT -- 1975 01:23:58,341 --> 01:23:59,776 ASSOCIATED WITH LEUKEMIA, SO I 1976 01:23:59,776 --> 01:24:01,010 THINK IT'S REALLY ABOUT 1977 01:24:01,010 --> 01:24:01,844 INCREASING AWARENESS. 1978 01:24:01,844 --> 01:24:03,146 I KNOW NHLBI IS PUTTING A LOT OF 1979 01:24:03,146 --> 01:24:04,247 FUNDING INTO TRYING TO DO THAT, 1980 01:24:04,247 --> 01:24:07,817 TO REALLY TRY TO -- ONE PATIENT, 1981 01:24:07,817 --> 01:24:14,857 HE WOULDN'T TAKE HYDROXYUREA -- 1982 01:24:14,857 --> 01:24:15,725 STARTED IT RIGHT AWAY. 1983 01:24:15,725 --> 01:24:18,127 SO I THINK IT'S REALLY ABOUT 1984 01:24:18,127 --> 01:24:19,562 EDUCATION NOT ONLY OF THE 1985 01:24:19,562 --> 01:24:20,830 PATIENTS BUT ALSO THE PROVIDERS 1986 01:24:20,830 --> 01:24:23,499 AND GETTING THE -- GETTING MORE 1987 01:24:23,499 --> 01:24:25,668 PEOPLE INTERESTED IN SICKLE 1988 01:24:25,668 --> 01:24:31,574 CELL --. 1989 01:24:31,574 --> 01:24:33,876 >> ANY OTHER QUESTIONS FOR 1990 01:24:33,876 --> 01:24:34,210 DR. FITZHUGH? 1991 01:24:34,210 --> 01:24:37,013 >> CAN I ASK A QUICK QUESTION, I 1992 01:24:37,013 --> 01:24:37,780 APOLOGIZE IF THIS WAS MY 1993 01:24:37,780 --> 01:24:38,348 IGNORANCE. 1994 01:24:38,348 --> 01:24:39,549 THIS WAS REALLY INTERESTING, A 1995 01:24:39,549 --> 01:24:42,151 GREAT BO DEAL BODY OF WORK. 1996 01:24:42,151 --> 01:24:43,119 THANK YOU. 1997 01:24:43,119 --> 01:24:46,089 SO THE MOST COMMON DRIVERS OF, 1998 01:24:46,089 --> 01:24:51,761 YOU KNOW, HEMATOPOEISIS ARE -- 1999 01:24:51,761 --> 01:24:53,830 AND DOSE THREE. 2000 01:24:53,830 --> 01:24:57,667 AND IT SEEMS LIKE THE HYPOTHESIS 2001 01:24:57,667 --> 01:25:00,937 IS THAT THE STRESS OF 2002 01:25:00,937 --> 01:25:02,038 HEMATOPOEISIS, YOU KNOW, YOU 2003 01:25:02,038 --> 01:25:08,278 WOULD HAVE TO -- ADVANTAGEOUS 2004 01:25:08,278 --> 01:25:10,113 PROLIFERATION, BUT WHY IS IT, 2005 01:25:10,113 --> 01:25:13,783 YOU KNOW, A DIFFERENT DRIVER IN 2006 01:25:13,783 --> 01:25:17,420 SICKLE CELL DISEASE? 2007 01:25:17,420 --> 01:25:20,023 WHAWHY IS IT NOT WHAT IS IN THE 2008 01:25:20,023 --> 01:25:20,657 COMMON POPULATION? 2009 01:25:20,657 --> 01:25:23,326 >> I SHOWED YOU THE TWO PATIENTS 2010 01:25:23,326 --> 01:25:28,464 I HAD TP53 MUTATIONS, MOTORS 2011 01:25:28,464 --> 01:25:29,766 COMMON WE FOUND WAS -- IT IS 2012 01:25:29,766 --> 01:25:30,867 STILL COMMON IN SICKLE CELL BUT 2013 01:25:30,867 --> 01:25:34,037 THE FREQUENCY OF TNA DAMAGE 2014 01:25:34,037 --> 01:25:39,842 RESPONSE GENES CAUSING TP53, 2015 01:25:39,842 --> 01:25:41,244 TP1MD IS MORE COMMON IN PATIENTS 2016 01:25:41,244 --> 01:25:42,011 WITH SICKLE CELL DISEASE AND WE 2017 01:25:42,011 --> 01:25:43,746 DON'T KNOW EXACTLY WHY. 2018 01:25:43,746 --> 01:25:45,481 IT'S ALMOST LIKE -- EVEN 2019 01:25:45,481 --> 01:25:46,816 PATIENTS THAT AREN'T GETTING 2020 01:25:46,816 --> 01:25:47,884 TRANSPLANTED, THEY CAN 2021 01:25:47,884 --> 01:25:49,886 DEVELOP -- LOOKS LIKE 2022 01:25:49,886 --> 01:25:50,620 THERAPY-ASSOCIATED LEUKEMIA. 2023 01:25:50,620 --> 01:25:52,689 SO THERE'S SOMETHING ABOUT THEIR 2024 01:25:52,689 --> 01:25:54,757 BONE MARROW -- THERE'S SOMETHING 2025 01:25:54,757 --> 01:25:55,658 ABOUT -- INFLAMMATION OR 2026 01:25:55,658 --> 01:25:57,627 WHATEVER THAT'S LEADING TO 2027 01:25:57,627 --> 01:25:58,728 THE -- SOMETHING THAT WE'RE 2028 01:25:58,728 --> 01:25:59,228 STUDYING NOW. 2029 01:25:59,228 --> 01:26:01,998 >> SO TP53 IS MORE COMMON IN THE 2030 01:26:01,998 --> 01:26:03,966 SICKLE CELL MUTATION AND SICKLE 2031 01:26:03,966 --> 01:26:06,269 CELL POPULATION THAN BEFORE? 2032 01:26:06,269 --> 01:26:08,371 >> MORE COMMON THAN WE EXPECTED, 2033 01:26:08,371 --> 01:26:08,705 THAT'S RIGHT. 2034 01:26:08,705 --> 01:26:12,175 >> OKAY, THANK YOU. 2035 01:26:12,175 --> 01:26:13,943 >> ANY OTHER QUESTIONS FOR 2036 01:26:13,943 --> 01:26:19,015 DR. FITZHUGH? 2037 01:26:19,015 --> 01:26:20,450 I DON'T SEE ANY. 2038 01:26:20,450 --> 01:26:21,651 THANK YOU VERY MUCH FOR A 2039 01:26:21,651 --> 01:26:22,118 WONDERFUL TALK. 2040 01:26:22,118 --> 01:26:28,791 [APPLAUSE] 2041 01:26:28,791 --> 01:26:30,259 WE'LL NOW TAKE A 30-MINUTE BREAK 2042 01:26:30,259 --> 01:26:31,761 SO YOU CAN WALK ACROSS THE HALL 2043 01:26:31,761 --> 01:26:33,796 AND GET SOME LUNCH. 2044 01:26:33,796 --> 01:26:36,324 WE'LL RECONVENE AT ABOUT 10:01. 2045 02:08:01,114 --> 01:26:39,879 >> ALL RIGHTY. 2046 01:26:39,879 --> 01:26:40,680 WELCOME BACK. 2047 01:26:40,680 --> 01:26:48,087 HOPE YOU ENJOYED THAT BREAK. 2048 01:26:48,087 --> 01:26:50,290 WE NOW MOVE TO THE FINAL AGENDA 2049 01:26:50,290 --> 01:26:52,892 ITEM ON TODAY'S MEETING, FOR 2050 01:26:52,892 --> 01:26:55,828 TODAY'S MEETING, VOTING ON THE 2051 01:26:55,828 --> 01:27:00,633 CONCEPTS FOR THIS ROUND. 2052 01:27:00,633 --> 01:27:01,634 HOWEVER, BEFORE WE PROCEED, I 2053 01:27:01,634 --> 01:27:03,503 HAVE SOME REMINDERS. 2054 01:27:03,503 --> 01:27:07,440 FOR DECISION LENS TECHNICAL 2055 01:27:07,440 --> 01:27:08,641 ISSUES, WHEN YOU'RE LOOKING AT 2056 01:27:08,641 --> 01:27:11,044 THE CONCEPTS AND VOTING, FOR ANY 2057 01:27:11,044 --> 01:27:20,353 TECHNICAL ISSUES, WE HAVE KAANAN 2058 01:27:20,353 --> 01:27:21,788 HERE FROM DECISION LENS AND 2059 01:27:21,788 --> 01:27:23,623 AFTER HE PERFORMS THE LIVE DEMO, 2060 01:27:23,623 --> 01:27:25,258 HE WILL BE HERE TO LEND 2061 01:27:25,258 --> 01:27:27,694 ASSISTANCE SHOULD YOU NEED THAT. 2062 01:27:27,694 --> 01:27:32,598 BRIAN GLEASON WILL PROVIDE 2063 01:27:32,598 --> 01:27:37,937 BACKUP SUPPORT AND FOR MORE 2064 01:27:37,937 --> 01:27:39,272 TECHNICAL SUPPORT WHICH IS 2065 01:27:39,272 --> 01:27:40,573 ONLINE, SO YOU SHOULD HAVE THOSE 2066 01:27:40,573 --> 01:27:43,109 EMAIL ADDRESSES. 2067 01:27:43,109 --> 01:27:44,077 THEY WILL BE DISPLAYED ON THE 2068 01:27:44,077 --> 01:27:46,079 SCREEN WHEN YOU PULL UP THE 2069 01:27:46,079 --> 01:27:48,781 APPLICATION, SO IF YOU NEED 2070 01:27:48,781 --> 01:27:52,385 TECHNICAL SUPPORT, YOU CAN TRY 2071 01:27:52,385 --> 01:27:54,687 CANAAN AND IF HE CAN'T HELP YOU, 2072 01:27:54,687 --> 01:27:56,022 THEN THEY'LL FORWARD ON TO THE 2073 01:27:56,022 --> 01:27:57,757 TECH ONLINE. 2074 01:27:57,757 --> 01:27:59,826 WITH THAT, I'LL TURN IT OVER TO 2075 01:27:59,826 --> 01:28:10,169 CANAAN FOR THE DEMO. 2076 01:28:15,675 --> 01:28:16,909 >> ALL RIGHT, GREAT. 2077 01:28:16,909 --> 01:28:18,244 HI, EVERYBODY. 2078 01:28:18,244 --> 01:28:20,446 I'M CANAAN GABLEY FROM DECISION 2079 01:28:20,446 --> 01:28:20,646 LENS. 2080 01:28:20,646 --> 01:28:23,583 THANK YOU ALL FOR HAVING YOU US 2081 01:28:23,583 --> 01:28:23,883 BACK. 2082 01:28:23,883 --> 01:28:26,986 WI'LL BE TAKING YOU THROUGH THE 2083 01:28:26,986 --> 01:28:28,388 DEMONSTRATION OF DECISION LENS. 2084 01:28:28,388 --> 01:28:29,589 WE'LL ALSO BE HERE ALL DAY IN 2085 01:28:29,589 --> 01:28:30,623 CASE YOU HAVE ANY QUESTIONS OR 2086 01:28:30,623 --> 01:28:33,960 RUN INTO ANY TECHNICAL ISSUES. 2087 01:28:33,960 --> 01:28:36,496 SO FOR THOSE THAT WILL BE 2088 01:28:36,496 --> 01:28:37,997 PARTICIPATING IN THE VOTING, YOU 2089 01:28:37,997 --> 01:28:39,899 SHOULD HAVE RECEIVED AN EMAIL 2090 01:28:39,899 --> 01:28:41,968 FROM 2091 01:28:41,968 --> 01:28:42,602 ADMINISTRATOR@DECISIONLENS.COM. 2092 01:28:42,602 --> 01:28:43,536 YOU CAN USE THE INSTRUCTIONS IN 2093 01:28:43,536 --> 01:28:46,572 THAT EMAIL TO DIRECTLY ACCESS 2094 01:28:46,572 --> 01:28:49,208 DECISION LENS AND GET LOGGED IN. 2095 01:28:49,208 --> 01:28:51,177 SO ONCE YOU ACCESS DECISION 2096 01:28:51,177 --> 01:28:55,081 LENS, YOU'LL COME TO A LOGIN 2097 01:28:55,081 --> 01:28:58,584 PAGE, YOU NEED YOUR EMAIL AND 2098 01:28:58,584 --> 01:28:59,685 PASSWORD IN ORDER TO GET IN. 2099 01:28:59,685 --> 01:29:02,955 THERE IS A FORGOT PASSWORD 2100 01:29:02,955 --> 01:29:04,624 OPTION ON THE SCREEN TO MANUALLY 2101 01:29:04,624 --> 01:29:05,925 RESET IT OR IF YOU NEED MORE 2102 01:29:05,925 --> 01:29:07,260 HELP, LET US KNOW AND WE'LL COME 2103 01:29:07,260 --> 01:29:07,560 HELP. 2104 01:29:07,560 --> 01:29:10,096 ONCE YOU GET LOGGED IN, YOU'LL 2105 01:29:10,096 --> 01:29:12,799 SEE WHAT IS DISPLAYED ON THE 2106 01:29:12,799 --> 01:29:14,434 ZOOM AND ALSO ON THE TOP OF THE 2107 01:29:14,434 --> 01:29:15,701 SCREEN HERE, THERE'S GOING TO BE 2108 01:29:15,701 --> 01:29:18,271 ONE TASK CALLED RATE 2109 01:29:18,271 --> 01:29:20,339 ALTERNATIVES FOR THE PORTFOLIO 2110 01:29:20,339 --> 01:29:22,642 TITLED IDP JUNE 2024 COUNCIL. 2111 01:29:22,642 --> 01:29:24,510 ALL YOU'LL NEED TO DO IS CLICK 2112 01:29:24,510 --> 01:29:27,013 ON THE TASK AT THE TOP. 2113 01:29:27,013 --> 01:29:30,416 YOU'LL SEE THAT WE ARE RATING 15 2114 01:29:30,416 --> 01:29:31,951 CONCEPTS HERE, AND YOU'LL SEE 2115 01:29:31,951 --> 01:29:33,519 THEM IN ORDER ON THE MIDDLE OF 2116 01:29:33,519 --> 01:29:34,887 THE SCREEN. 2117 01:29:34,887 --> 01:29:36,422 SO TO BEGIN, WE'LL JUST HAVE YOU 2118 01:29:36,422 --> 01:29:38,491 CLICK ON 3938, WHICH IS THE 2119 01:29:38,491 --> 01:29:41,894 FIRST ONE. 2120 01:29:41,894 --> 01:29:44,197 AND THIS WILL BRING UP THE 2121 01:29:44,197 --> 01:29:44,831 CONCEPT. 2122 01:29:44,831 --> 01:29:46,799 SO YOU'LL SEE THE BLUE TEXT AT 2123 01:29:46,799 --> 01:29:47,633 THE TOP IS THE CONCEPT. 2124 01:29:47,633 --> 01:29:49,102 YOU WANT TO GET A LITTLE BIT 2125 01:29:49,102 --> 01:29:51,904 MORE INFORMATION ON THE CONCEPT, 2126 01:29:51,904 --> 01:29:54,040 AND THE DETAILS BEHIND IT, YOU 2127 01:29:54,040 --> 01:29:55,575 CAN DO THAT BY CLICKING AT THE 2128 01:29:55,575 --> 01:29:57,743 BLUE TEXT ON THE TOP, THE 2129 01:29:57,743 --> 01:29:58,311 CONCEPT TITLE. 2130 01:29:58,311 --> 01:30:00,480 YOU'LL SEE THAT WILL OPEN UP THE 2131 01:30:00,480 --> 01:30:01,013 SIDECAR HERE. 2132 01:30:01,013 --> 01:30:04,417 YOU CAN SCROLL, READ ALL YOU 2133 01:30:04,417 --> 01:30:04,750 NEED. 2134 01:30:04,750 --> 01:30:06,252 THEN YOU'LL PROCEED TO WORK DOWN 2135 01:30:06,252 --> 01:30:07,186 THE PAGE. 2136 01:30:07,186 --> 01:30:09,355 SO THE PURPLE TEXT ARE THE 2137 01:30:09,355 --> 01:30:10,690 CRITERIA THAT YOU'LL BE 2138 01:30:10,690 --> 01:30:11,657 EVALUATING THE CONCEPT AGAINST. 2139 01:30:11,657 --> 01:30:13,292 SO THE FIRST ONE BEING 2140 01:30:13,292 --> 01:30:17,330 IMPORTANCE OF THE SCIENTIFIC, 2141 01:30:17,330 --> 01:30:20,099 MEDICAL, PUBLIC HEALTH OR 2142 01:30:20,099 --> 01:30:22,168 RESEARCH-RELATED QUESTIONS 2143 01:30:22,168 --> 01:30:22,502 OPPORTUNITY. 2144 01:30:22,502 --> 01:30:24,470 IF YOU CLICK ON THE PURPLE TEXT, 2145 01:30:24,470 --> 01:30:26,372 YOU'LL SEE THE DESCRIPTION ON 2146 01:30:26,372 --> 01:30:26,806 THE RIGHT-HAND SIDE. 2147 01:30:26,806 --> 01:30:28,207 IF YOU WANT TO READ A LITTLE BIT 2148 01:30:28,207 --> 01:30:30,042 MORE, JUST CLICK THE "MORE" 2149 01:30:30,042 --> 01:30:31,043 OPTION AND YOU'LL SEE THAT 2150 01:30:31,043 --> 01:30:31,344 EXPANDS. 2151 01:30:31,344 --> 01:30:32,879 THEN FOR EACH OF THE RATING 2152 01:30:32,879 --> 01:30:34,881 SCALE OPTIONS FROM EXTREMELY 2153 01:30:34,881 --> 01:30:37,583 IMPORTANT TO NOT IMPORTANT, 2154 01:30:37,583 --> 01:30:39,886 YOU'LL SEE THE DEFINITIONS FOR 2155 01:30:39,886 --> 01:30:41,521 EACH ON THE RIGHT-HAND SIDE. 2156 01:30:41,521 --> 01:30:42,755 SO NOW THAT YOU'VE READ 2157 01:30:42,755 --> 01:30:44,257 EVERYTHING YOU NEED TO EVALUATE 2158 01:30:44,257 --> 01:30:46,359 THE CONCEPT AGAINST THIS 2159 01:30:46,359 --> 01:30:47,760 SPECIFIC CRITERION, YOU CAN GO 2160 01:30:47,760 --> 01:30:50,396 AHEAD AND MAKE YOUR JUDGMENT. 2161 01:30:50,396 --> 01:30:59,138 I THINK IT WOULD BE VERY -- IF 2162 01:30:59,138 --> 01:31:00,673 FOR WHATEVER REASON YOU LOSE 2163 01:31:00,673 --> 01:31:02,775 INTERNET, GET LOGGED OUT, 2164 01:31:02,775 --> 01:31:05,711 EVERYTHING WILL BE AUTO SAVED 2165 01:31:05,711 --> 01:31:06,913 AND YOUR PROGRESS WILL BE 2166 01:31:06,913 --> 01:31:07,446 TRACKED. 2167 01:31:07,446 --> 01:31:09,649 SO GO DOWN THE PAGE FOR EACH 2168 01:31:09,649 --> 01:31:10,516 CRITERION, MAKING YOUR 2169 01:31:10,516 --> 01:31:10,950 ASSESSMENT. 2170 01:31:10,950 --> 01:31:12,084 THEN ONCE YOU REACH THE BOTTOM 2171 01:31:12,084 --> 01:31:14,554 OF THE PAGE, YOU WILL SEE A 2172 01:31:14,554 --> 01:31:15,755 "NEXT" BUTTON ON THE SCREEN. 2173 01:31:15,755 --> 01:31:17,790 ONCE YOU'VE COMPLETED ALL SIX 2174 01:31:17,790 --> 01:31:19,525 EVALUATIONS FOR THE CONCEPT, 2175 01:31:19,525 --> 01:31:20,927 THAT WILL LIGHT UP IN ORANGE. 2176 01:31:20,927 --> 01:31:22,662 AND YOU CAN CLICK THE NEXT 2177 01:31:22,662 --> 01:31:24,197 BUTTON AND IT WILL BRING UP THE 2178 01:31:24,197 --> 01:31:25,531 NEXT CONCEPT. 2179 01:31:25,531 --> 01:31:28,534 ONE OTHER PIECE I DO WANT TO 2180 01:31:28,534 --> 01:31:30,436 POINT OUT, IF YOU HAVE ANY 2181 01:31:30,436 --> 01:31:31,637 COMMENTS THAT YOU WOULD LIKE TO 2182 01:31:31,637 --> 01:31:33,172 LEAVE IN THE TOOL, THIS IS VERY 2183 01:31:33,172 --> 01:31:37,877 HELPFUL AFTER THE FACT, SO YOU 2184 01:31:37,877 --> 01:31:40,246 CAN LOOK AT THEM IN A SINGLE 2185 01:31:40,246 --> 01:31:40,479 OUTPUT. 2186 01:31:40,479 --> 01:31:42,548 IF YOU HAD A COMMENT, A 2187 01:31:42,548 --> 01:31:44,016 QUESTION, A CONCERN, ANYTHING 2188 01:31:44,016 --> 01:31:45,284 THAT YOU WOULD LIKE TO RECORD IN 2189 01:31:45,284 --> 01:31:47,587 THE TOOL, YOU CAN USE THE SPEECH 2190 01:31:47,587 --> 01:31:49,222 BUBBLE OR THIS COMMENT BUBBLE 2191 01:31:49,222 --> 01:31:51,958 THAT'S LOCATED RIGHT NEXT TO THE 2192 01:31:51,958 --> 01:31:52,391 CRITERIA NAME. 2193 01:31:52,391 --> 01:31:56,028 WHEN YOU CLICK THAT, THE SIDEBAR 2194 01:31:56,028 --> 01:31:57,330 WILL OPEN UP AND YOU'LL BE ABLE 2195 01:31:57,330 --> 01:31:58,631 TO ENTER IN YOUR COMMENTS HERE. 2196 01:31:58,631 --> 01:32:00,166 SO I'M JUST GOING TO TYPE IN A 2197 01:32:00,166 --> 01:32:01,267 TEST COMMENT. 2198 01:32:01,267 --> 01:32:03,502 ONCE YOU'RE READY TO PUBLISH AND 2199 01:32:03,502 --> 01:32:05,204 ENTER THIS COMMENT INTO THE 2200 01:32:05,204 --> 01:32:07,940 TOOL, ENTER THE -- PRESS THE 2201 01:32:07,940 --> 01:32:11,010 ORANGE "ADD COMMENT" BUTTON AND 2202 01:32:11,010 --> 01:32:15,915 YOU'LL SEE THAT. 2203 01:32:15,915 --> 01:32:17,450 YOU WILL BE ABLE TO SEE YOUR 2204 01:32:17,450 --> 01:32:19,518 PROGRESS IN THE TOP RIGHT, SO AS 2205 01:32:19,518 --> 01:32:21,487 WE WORK OUR WAY THROUGH, YOU'LL 2206 01:32:21,487 --> 01:32:22,822 SEE THE PROGRESS IN THE TOP 2207 01:32:22,822 --> 01:32:23,689 RIGHT. 2208 01:32:23,689 --> 01:32:24,991 MARA AND I WILL ALSO BE 2209 01:32:24,991 --> 01:32:25,992 MONITORING THAT TO MAKE SURE 2210 01:32:25,992 --> 01:32:27,093 THAT EVERYONE IS STAYING ON 2211 01:32:27,093 --> 01:32:27,660 PACE. 2212 01:32:27,660 --> 01:32:35,067 BUT THAT SHOULD BE -- ANY 2213 01:32:35,067 --> 01:32:36,936 QUESTIONS OR ANYTHING THAT YOU'D 2214 01:32:36,936 --> 01:32:39,772 LIKE ME TO RE-EMPHASIZE? 2215 01:32:39,772 --> 01:32:40,773 OKAY. 2216 01:32:40,773 --> 01:32:42,141 WE'LL BE OVER HERE, SO IF YOU 2217 01:32:42,141 --> 01:32:43,376 HAVE ANY ISSUES GETTING IN, 2218 01:32:43,376 --> 01:32:44,577 PLEASE LET US KNOW. 2219 01:32:44,577 --> 01:32:46,445 AND WE'LL COME BY AND ASSIST. 2220 01:32:46,445 --> 01:32:48,881 THANK YOU. 2221 01:32:48,881 --> 01:32:50,616 >> OKAY. 2222 01:32:50,616 --> 01:32:52,151 THE LUNG DIVISION IS GOING TO 2223 01:32:52,151 --> 01:32:54,453 START US OFF. 2224 01:32:54,453 --> 01:32:55,421 JIM, ANY TIME YOU'RE READY. 2225 01:32:55,421 --> 01:32:56,622 >> OKAY, VERY GOOD. 2226 01:32:56,622 --> 01:32:57,823 SO SOUNDS LIKE YOU ALREADY DID 2227 01:32:57,823 --> 01:32:58,958 THE FIRST ONE, SO WE CAN MOVE 2228 01:32:58,958 --> 01:33:00,326 ON. 2229 01:33:00,326 --> 01:33:02,728 NO, WE'RE GOING TO START WITH -- 2230 01:33:02,728 --> 01:33:03,496 WE HAVE FOUR. 2231 01:33:03,496 --> 01:33:05,464 THE FIRST TWO ARE A PACKAGE SO 2232 01:33:05,464 --> 01:33:05,798 REALLY THREE. 2233 01:33:05,798 --> 01:33:09,468 WE'RE GOING TO DO INITIATIVE 2234 01:33:09,468 --> 01:33:12,071 NUMBER 3938 AND 3940. 2235 01:33:12,071 --> 01:33:14,573 THIS IS ENTITLED VIRAL 2236 01:33:14,573 --> 01:33:17,343 INFECTIONS IN YOUNG -- IN THE 2237 01:33:17,343 --> 01:33:18,544 YOUNG LUNG. 2238 01:33:18,544 --> 01:33:21,147 THE FINAL CLINICAL CONSORTIUM 2239 01:33:21,147 --> 01:33:22,348 AND THE DATA COORDINATING 2240 01:33:22,348 --> 01:33:22,682 CENTER. 2241 01:33:22,682 --> 01:33:26,419 SO AS I SAID, THESE ARE A 2242 01:33:26,419 --> 01:33:28,054 PACKAGE, TWO GO TOGETHER, IT'S 2243 01:33:28,054 --> 01:33:29,989 THE COORDINATING CENTER AND THE 2244 01:33:29,989 --> 01:33:30,990 CLINICAL COORDINATING CENTERS. 2245 01:33:30,990 --> 01:33:32,291 SO THIS WOULD BE A NEW 2246 01:33:32,291 --> 01:33:34,060 CONSORTIUM THAT WE ARE PROPOSING 2247 01:33:34,060 --> 01:33:35,594 THAT WE BUILD THAT WILL 2248 01:33:35,594 --> 01:33:37,997 ELUCIDATE THE VIRAL LOWER 2249 01:33:37,997 --> 01:33:39,298 RESPIRATORY TRACT INFECTIONS IN 2250 01:33:39,298 --> 01:33:42,702 THE YOUNG LUNG, OTHERWISE KNOWN 2251 01:33:42,702 --> 01:33:43,235 AS VINYL. 2252 01:33:43,235 --> 01:33:45,004 THIS IS A DEEP PHENOTYPING 2253 01:33:45,004 --> 01:33:47,073 EFFORT TO UNDERSTAND BOTH THE 2254 01:33:47,073 --> 01:33:50,042 CLINICAL, CELLULAR AND MOLECULAR 2255 01:33:50,042 --> 01:33:54,413 LEVEL OF WHAT IS GOING ON IN 2256 01:33:54,413 --> 01:33:55,948 ABOUT 1500 KIDS, NOT QUITE A 2257 01:33:55,948 --> 01:33:58,417 BIRTH COHORT BUT CLOSE TO IT, 2258 01:33:58,417 --> 01:34:00,920 ZERO TO 2-YEAR-OLD 2259 01:34:00,920 --> 01:34:02,088 INFANTS/CHILDREN WHO ARE 2260 01:34:02,088 --> 01:34:03,522 HOSPITALIZED WITH VIRAL LOWER 2261 01:34:03,522 --> 01:34:04,724 RESPIRATORY TRACT INFECTIONS. 2262 01:34:04,724 --> 01:34:06,926 AND TO FOLLOW THESE YOUNG 2263 01:34:06,926 --> 01:34:09,328 CHILDREN UNTIL PRESCHOOL AGE TO 2264 01:34:09,328 --> 01:34:11,864 LOOK AT PULMONARY SEQUELAE. 2265 01:34:11,864 --> 01:34:13,065 SO I DON'T HAVE TO TELL THIS 2266 01:34:13,065 --> 01:34:14,500 GROUP THAT VIRAL LOWER 2267 01:34:14,500 --> 01:34:15,801 RESPIRATORY TRACT INFECTIONS 2268 01:34:15,801 --> 01:34:26,879 SUCH AS BRONG BRO -- THIS LEAD, 2269 01:34:27,246 --> 01:34:28,614 MANY HOSPITALIZATIONS IN THE 2270 01:34:28,614 --> 01:34:31,450 UNITED STATES. 2271 01:34:31,450 --> 01:34:34,620 ALSO THIS POPULATION 2272 01:34:34,620 --> 01:34:36,055 DISPROPORTIONATELY AFFECTS YOUNG 2273 01:34:36,055 --> 01:34:38,224 CHILDREN, THOSE BORN SOMETIMES 2274 01:34:38,224 --> 01:34:40,092 PREMATURE, ALSO THOSE IN LOW 2275 01:34:40,092 --> 01:34:42,828 SOCIOECONOMIC CIRCUMSTANCES. 2276 01:34:42,828 --> 01:34:44,497 SO I THINK THIS IS A VERY 2277 01:34:44,497 --> 01:34:47,266 IMPORTANT INITIATIVE TO BEGIN TO 2278 01:34:47,266 --> 01:34:48,534 LOOK AT THE PROBLEM BECAUSE 2279 01:34:48,534 --> 01:34:50,369 RIGHT NOW, THERE ARE NO NEW 2280 01:34:50,369 --> 01:34:51,604 THERAPIES TO TREAT. 2281 01:34:51,604 --> 01:34:53,906 MOST OF THE MANIFESTATIONS, 2282 01:34:53,906 --> 01:34:58,010 WHETHER IT'S BRONCHIOLITIS OR 2283 01:34:58,010 --> 01:35:00,379 OTHER THINGS THAT RESULT FROM 2284 01:35:00,379 --> 01:35:03,983 THESE LOWER RESPIRATORY TRACT 2285 01:35:03,983 --> 01:35:04,650 INFECTIONS -- FOR MANY, MANY 2286 01:35:04,650 --> 01:35:05,217 YEARS. 2287 01:35:05,217 --> 01:35:07,987 THE CLINICAL COURSE IS VERY 2288 01:35:07,987 --> 01:35:09,188 VARIED, THE RESPONSE TO THERAPY 2289 01:35:09,188 --> 01:35:11,056 IS ALSO QUITE VARIED, AND 2290 01:35:11,056 --> 01:35:12,324 PARTICULARLY WHERE THERE'S 2291 01:35:12,324 --> 01:35:14,660 SEVERE INFECTION, AND THESE 2292 01:35:14,660 --> 01:35:17,396 CERTAINLY HAVE A POWERFUL IMPACT 2293 01:35:17,396 --> 01:35:19,832 ON EARLY INSULTS TO THE YOUNG 2294 01:35:19,832 --> 01:35:24,170 LUNG, AND THEN ON TO AUTOGENY OF 2295 01:35:24,170 --> 01:35:26,272 THE RESPIRATORY AMINO PHENOTYPE 2296 01:35:26,272 --> 01:35:29,408 THAT MAY IMPACT ON FUTURE LUNG 2297 01:35:29,408 --> 01:35:30,209 AND AIRWAY HEALTH. 2298 01:35:30,209 --> 01:35:32,077 AND AGAIN, THAT SORT OF ROLLS ON 2299 01:35:32,077 --> 01:35:34,413 INTO ALL OF THE ISSUES WE SEE 2300 01:35:34,413 --> 01:35:36,115 WITH THE PEDIATRIC POPULATION, 2301 01:35:36,115 --> 01:35:38,417 WHETHER IT'S PEDIATRIC ASTHMA OR 2302 01:35:38,417 --> 01:35:41,086 OTHER KINDS OF ALLERGIC AND 2303 01:35:41,086 --> 01:35:42,054 IMMUNOLOGICAL CONDITIONS 2304 01:35:42,054 --> 01:35:43,122 AFFECTING THE YOUNG LUNG. 2305 01:35:43,122 --> 01:35:44,890 AND THIS CONTINUES SORT OF 2306 01:35:44,890 --> 01:35:46,859 UNABATED, REALLY, IN MANY WAYS 2307 01:35:46,859 --> 01:35:49,395 BECAUSE OF THE LACK OF REAL GOOD 2308 01:35:49,395 --> 01:35:51,697 WAYS TO MITIGATE AT THE EARLIEST 2309 01:35:51,697 --> 01:35:52,331 STAGE. 2310 01:35:52,331 --> 01:35:57,470 SO THIS PARTICULAR INITIATIVE 2311 01:35:57,470 --> 01:35:59,238 WILL ESTABLISH CLINICAL CENTERS, 2312 01:35:59,238 --> 01:36:01,640 COORDINATING CENTERS. 2313 01:36:01,640 --> 01:36:05,711 IT WILL ESSENTIALLY ENROLL THIS 2314 01:36:05,711 --> 01:36:09,348 COHORT THAT WE HOPE THAT WE CAN 2315 01:36:09,348 --> 01:36:12,184 DO HAVE, VERY EXTENSIVE DEPUTY 2316 01:36:12,184 --> 01:36:15,454 KNOW TYPING ON, PARTICULARLY THE 2317 01:36:15,454 --> 01:36:16,021 IMMUNOPHENOTYPING WHICH WE 2318 01:36:16,021 --> 01:36:16,822 BELIEVE IS SOMEWHAT LACKING 2319 01:36:16,822 --> 01:36:17,556 RIGHT NOW. 2320 01:36:17,556 --> 01:36:18,958 NOT ONLY WILL WE UNDERSTAND A 2321 01:36:18,958 --> 01:36:21,360 BIT MORE ABOUT BEST THERAPIES 2322 01:36:21,360 --> 01:36:25,598 AND HOW TO BEST INTERVENE IN 2323 01:36:25,598 --> 01:36:26,999 THESE KIDS, BUT ALSO TO BEGIN TO 2324 01:36:26,999 --> 01:36:30,503 REALLY PUT THE PIECES TOGETHER 2325 01:36:30,503 --> 01:36:33,572 THAT GO FROM INITIAL INSULT, 2326 01:36:33,572 --> 01:36:34,840 PATHOLOGY, TO POTENTIAL 2327 01:36:34,840 --> 01:36:37,610 COMPROMISE IN THAT LUNG AS THEY 2328 01:36:37,610 --> 01:36:40,613 AGE, AND AS THEY DEVELOP. 2329 01:36:40,613 --> 01:36:41,647 AND THIS KIND OF PULLS ALL OF 2330 01:36:41,647 --> 01:36:42,915 THAT TOGETHER IN A WAY THAT WE 2331 01:36:42,915 --> 01:36:45,451 HOPE, OVER TIME, THAT WE'LL 2332 01:36:45,451 --> 01:36:46,986 BEGIN TO LOOK -- AND YOU HEARD 2333 01:36:46,986 --> 01:36:48,821 EARLY, HOW CAN WE INTERVENE 2334 01:36:48,821 --> 01:36:49,255 EARLY? 2335 01:36:49,255 --> 01:36:50,689 WE CAN'T GET MUCH EARLIER THAN 2336 01:36:50,689 --> 01:36:52,124 THIS POPULATION, SO THIS IS A 2337 01:36:52,124 --> 01:36:54,860 WAY IN WHICH WE CAN REALLY START 2338 01:36:54,860 --> 01:36:56,395 TO REALLY PUT THE PIECES 2339 01:36:56,395 --> 01:36:58,063 TOGETHER. 2340 01:36:58,063 --> 01:37:00,799 WE THINK THIS WOULD BE REALLY A 2341 01:37:00,799 --> 01:37:03,302 GREAT START TO NOT ONLY 2342 01:37:03,302 --> 01:37:05,137 UNDERSTAND BETTER THIS CONDITION 2343 01:37:05,137 --> 01:37:07,039 IN YOUNG CHILDREN, ALSO 2344 01:37:07,039 --> 01:37:08,674 UNDERSTAND VIRAL RESPIRATORY 2345 01:37:08,674 --> 01:37:10,743 DISEASE, WHICH ALSO HAS, AS YOU 2346 01:37:10,743 --> 01:37:11,944 KNOW, RAMIFICATIONS OR 2347 01:37:11,944 --> 01:37:13,379 IMPLICATIONS FOR OTHER KINDS OF 2348 01:37:13,379 --> 01:37:14,513 RESPIRATORY INSULTS, WHETHER 2349 01:37:14,513 --> 01:37:16,348 IT'S VIRAL, BACTERIAL OR 2350 01:37:16,348 --> 01:37:16,882 OTHERWISE. 2351 01:37:16,882 --> 01:37:19,418 SO THIS IS A LARGE PROGRAM, IT'S 2352 01:37:19,418 --> 01:37:21,186 AMBITIOUS, IT'S ONE THAT TARGETS 2353 01:37:21,186 --> 01:37:25,558 A FRAGILE AND SOMEWHAT 2354 01:37:25,558 --> 01:37:27,393 UNDERREPRESENTED POPULATION. 2355 01:37:27,393 --> 01:37:28,727 IT'S AN IMPORTANT ONE FOR, I 2356 01:37:28,727 --> 01:37:31,130 THINK, US TO UNDERSTAND NOT ONLY 2357 01:37:31,130 --> 01:37:32,464 THE CLINICAL PROBLEM BUT ALSO 2358 01:37:32,464 --> 01:37:35,200 TRY TO UNTANGLE THE BIOLOGY AND 2359 01:37:35,200 --> 01:37:40,339 THE BASIC UNDERSTANDING OF HOW 2360 01:37:40,339 --> 01:37:41,874 THE YOUNG LUNG RESPONDS TO THESE 2361 01:37:41,874 --> 01:37:44,076 INSULTS AND HOW WE CAN GET IN, 2362 01:37:44,076 --> 01:37:44,977 UNDERSTAND IT BETTER AND LOOK 2363 01:37:44,977 --> 01:37:46,712 FOR WAYS TO BETTER TREAT IT. 2364 01:37:46,712 --> 01:37:48,247 SO WITH THAT, I'LL STOP. 2365 01:37:48,247 --> 01:37:50,516 HAPPY TO TRY TO TAKE QUESTIONS. 2366 01:37:50,516 --> 01:37:53,852 WE HAVE A NUMBER OF STAFF IN THE 2367 01:37:53,852 --> 01:37:55,287 DIVISION THAT HAVE BEEN INVOLVED 2368 01:37:55,287 --> 01:37:59,325 IN DEVELOPMENT OF THIS 2369 01:37:59,325 --> 01:37:59,858 INITIATIVE. 2370 01:37:59,858 --> 01:38:03,262 DRR NATAJARAN, OUR RESIDENT 2371 01:38:03,262 --> 01:38:04,263 PEDIATRIC INTENSIVIST, HAS BEEN 2372 01:38:04,263 --> 01:38:05,464 LEADING THIS EFFORT, CERTAINLY 2373 01:38:05,464 --> 01:38:06,699 CAN HELP ANSWER QUESTIONS IF I 2374 01:38:06,699 --> 01:38:06,966 CAN'T. 2375 01:38:06,966 --> 01:38:16,842 I'LL STOP THERE. 2376 01:38:16,842 --> 01:38:18,444 >> VERY IMPORTANT INITIATIVE. 2377 01:38:18,444 --> 01:38:20,079 OBVIOUSLY WE'LL BE STARTING 2378 01:38:20,079 --> 01:38:22,181 AFTER THE START OF RSV 2379 01:38:22,181 --> 01:38:23,382 PREVENTION, WHICH IS VERY LIKELY 2380 01:38:23,382 --> 01:38:26,785 TO CHANGE EPIDEMIOLOGY 2381 01:38:26,785 --> 01:38:27,753 TREMENDOUSLY, AND THE SECOND 2382 01:38:27,753 --> 01:38:28,954 COMMENT, I THINK IT'S GREAT AND 2383 01:38:28,954 --> 01:38:32,024 REALLY IMPORTANT THAT THE LONG 2384 01:38:32,024 --> 01:38:34,226 TERM OUTCOME OF ASTHMA BEING 2385 01:38:34,226 --> 01:38:35,327 FOLLOWED, BUT ONE OF THE THINGS 2386 01:38:35,327 --> 01:38:36,395 WE DON'T KNOW, THIS ASSOCIATION 2387 01:38:36,395 --> 01:38:38,530 HAS BEEN ESTABLISHED IN LIKE A 2388 01:38:38,530 --> 01:38:39,865 HUNDRED STUDIES, BUT ONE OF THE 2389 01:38:39,865 --> 01:38:41,100 THINGS WE REALLY DON'T KNOW THAT 2390 01:38:41,100 --> 01:38:42,468 I DIDN'T SEE SPECIFICALLY 2391 01:38:42,468 --> 01:38:43,569 ADDRESSED BUT I THINK COULD BE 2392 01:38:43,569 --> 01:38:45,004 VERY IMPORTANT IS REALLY 2393 01:38:45,004 --> 01:38:47,072 UNDERSTANDING THE SHARED GENETIC 2394 01:38:47,072 --> 01:38:48,073 PREDISPOSITION TO THESE TWO 2395 01:38:48,073 --> 01:38:50,042 DISEASES. 2396 01:38:50,042 --> 01:38:59,618 SEVERE BRONCHIOLITIS AND ASTHMA. 2397 01:38:59,618 --> 01:39:00,686 >> AGAIN, THESE ARE GREAT 2398 01:39:00,686 --> 01:39:01,453 COMMENTS AND CERTAINLY WE CAN 2399 01:39:01,453 --> 01:39:02,688 KIND OF BUILD THIS INTO IT, I 2400 01:39:02,688 --> 01:39:04,223 THINK IT LENDS ITSELF FOR DOING 2401 01:39:04,223 --> 01:39:07,092 THAT, FOR SURE. 2402 01:39:07,092 --> 01:39:07,860 YES. 2403 01:39:07,860 --> 01:39:11,263 >> I'M NOT A PEDIATRICIAN, BUT 2404 01:39:11,263 --> 01:39:13,999 AS AN ADULT PULMONOLOGIST, I 2405 01:39:13,999 --> 01:39:17,569 THINK IT IS INCREASINGLY 2406 01:39:17,569 --> 01:39:19,004 APPARENT HOW IMPORTANT CHILDHOOD 2407 01:39:19,004 --> 01:39:20,873 LUNG DISEASES ARE FOR THE 2408 01:39:20,873 --> 01:39:23,275 DEVELOPMENT OF CHRONIC LUNG 2409 01:39:23,275 --> 01:39:24,677 DISEASES. 2410 01:39:24,677 --> 01:39:26,211 AND UNDERSTANDING THE BASIC 2411 01:39:26,211 --> 01:39:27,446 MECHANISMS AND BEING ABLE TO 2412 01:39:27,446 --> 01:39:29,982 FOLLOW A COHORT OVER TIME IS 2413 01:39:29,982 --> 01:39:32,851 GOING TO BE CRITICALLY IMPORTANT 2414 01:39:32,851 --> 01:39:35,020 FOR INSIGHTS INTO COPD, ASTHMA 2415 01:39:35,020 --> 01:39:36,355 AND OTHER LUNG DISEASES. 2416 01:39:36,355 --> 01:39:37,556 SO I THINK THIS IS REALLY 2417 01:39:37,556 --> 01:39:38,424 IMPORTANT, AND I ALSO WOULD LOVE 2418 01:39:38,424 --> 01:39:42,828 TO SEE IT CONTINUE BEYOND THE 2419 01:39:42,828 --> 01:39:44,029 NEXT FIVE YEARS AND ONWARD. 2420 01:39:44,029 --> 01:39:47,966 >> YEAH, IT HAS TO. 2421 01:39:47,966 --> 01:39:50,269 I SAY THAT JOKINGLY, BUT WE 2422 01:39:50,269 --> 01:39:53,138 CAN'T EVEN PICK UP THAT ASTHMA 2423 01:39:53,138 --> 01:39:57,076 SIGNAL THAT IS SIGNIFICANT -- 2424 01:39:57,076 --> 01:39:58,243 CAN SPEAK TO THIS MUCH BETTER 2425 01:39:58,243 --> 01:39:59,912 THAN I CAN, UNTIL PROBABLY 2426 01:39:59,912 --> 01:40:02,548 THEY'RE PRESCHOOL AGE, 5, 7, 2427 01:40:02,548 --> 01:40:03,515 SOMETHING LIKE THAT, SO THIS HAS 2428 01:40:03,515 --> 01:40:04,917 TO GO A LITTLE BIT FURTHER IF 2429 01:40:04,917 --> 01:40:05,818 WE'RE GOING TO TRY TO PICK UP 2430 01:40:05,818 --> 01:40:07,152 SOME OF THOSE LONG-TERM SIGNALS 2431 01:40:07,152 --> 01:40:08,353 THAT I THINK ARE CRITICAL ALSO 2432 01:40:08,353 --> 01:40:10,222 FOR REALLY NAILING THIS DOWN, 2433 01:40:10,222 --> 01:40:15,461 WHAT DOES THAT INSULT DO TO THAT 2434 01:40:15,461 --> 01:40:16,695 DEVELOPMENT OF THAT LATER 2435 01:40:16,695 --> 01:40:16,962 SEQUELLA. 2436 01:40:16,962 --> 01:40:20,833 >> AND I DIDN'T READ CLOSE 2437 01:40:20,833 --> 01:40:22,067 ENOUGH, IS THERE ANY HEALTHY 2438 01:40:22,067 --> 01:40:22,901 COHORT CONTROL AS PART OF THIS? 2439 01:40:22,901 --> 01:40:24,670 >> WELL, THAT'S AN INCREDIBLY 2440 01:40:24,670 --> 01:40:26,138 IMPORTANT QUESTION, AND WE HAVE 2441 01:40:26,138 --> 01:40:27,439 BEEN WRESTLING WITH THAT ONE. 2442 01:40:27,439 --> 01:40:28,640 THE BOARD OF EXTERNAL EXPERTS 2443 01:40:28,640 --> 01:40:30,943 BROUGHT THAT UP TOO, A 2444 01:40:30,943 --> 01:40:32,711 CONTEMPORARY CONTROL FOR THESE 2445 01:40:32,711 --> 01:40:33,145 KIDS. 2446 01:40:33,145 --> 01:40:39,318 AND WE DO HAVE SOME LEAPS. 2447 01:40:39,318 --> 01:40:40,886 IT'S NOT BUILT IN TO HAVE A 2448 01:40:40,886 --> 01:40:41,653 CONTROL GROUP HERE. 2449 01:40:41,653 --> 01:40:43,288 THAT, WE'RE LOOKING AT BUT 2450 01:40:43,288 --> 01:40:44,356 OBVIOUSLY IT WOULD ADD A LOT 2451 01:40:44,356 --> 01:40:44,857 MORE TO IT. 2452 01:40:44,857 --> 01:40:46,391 BUT WE DO THINK THERE MAY BE 2453 01:40:46,391 --> 01:40:48,827 SOME OTHER COHORTS THAT EXIST 2454 01:40:48,827 --> 01:40:51,296 THAT COULD POTENTIALLY ALIGN, 2455 01:40:51,296 --> 01:40:55,267 PARTICULARLY THOSE THAT ARE 2456 01:40:55,267 --> 01:40:56,468 FUNDED BY THE NIAID, EVEN THOUGH 2457 01:40:56,468 --> 01:40:59,538 THEY ARE NOT IN-HOSPITAL-ROW 2458 01:40:59,538 --> 01:41:05,444 LATED E-RELATEDEVENTS OR KIDS TG 2459 01:41:05,444 --> 01:41:06,545 HOSPITALIZED. 2460 01:41:06,545 --> 01:41:07,880 THERE IS OUTPATIENT WORK THEY'RE 2461 01:41:07,880 --> 01:41:11,049 DOING THAT MIGHT ALSO GIVE US 2462 01:41:11,049 --> 01:41:12,251 SOME -- ECHO MIGHT BE ANOTHER 2463 01:41:12,251 --> 01:41:15,220 PLACE THAT WE CAN LOOK AT FOR 2464 01:41:15,220 --> 01:41:20,492 CONTROLS THAT AGAIN MAY ALIGN. 2465 01:41:20,492 --> 01:41:22,795 WE HEAR YOU, WE KNOW THAT'S A 2466 01:41:22,795 --> 01:41:23,896 PROBLEM, AN ISSUE, AND WE'RE 2467 01:41:23,896 --> 01:41:24,863 TRYING TO DO WHAT WE CAN TO MAKE 2468 01:41:24,863 --> 01:41:25,764 SURE WE HAVE SOMETHING TO 2469 01:41:25,764 --> 01:41:26,231 COMPARE TO. 2470 01:41:26,231 --> 01:41:29,701 >> I HAVE A QUESTION, ALIGNED 2471 01:41:29,701 --> 01:41:30,702 EXACTLY WITH THE CONTROL ISSUE. 2472 01:41:30,702 --> 01:41:32,671 I SAW THE PLAN IS TO USE OTHER 2473 01:41:32,671 --> 01:41:33,872 COHORTS WITH LESS SEVERE 2474 01:41:33,872 --> 01:41:35,307 DISEASE, WHICH I THINK WOULD BE 2475 01:41:35,307 --> 01:41:39,244 FINE, BUT ARE THEY EQUALLY AS 2476 01:41:39,244 --> 01:41:40,646 TYPICALLY CHARACTERIZED AS THIS 2477 01:41:40,646 --> 01:41:42,447 COHORT IS OR WOULD THEY BE 2478 01:41:42,447 --> 01:41:44,316 ESPECIALLY IN TERMS OF LIKE 2479 01:41:44,316 --> 01:41:45,184 GENETIC PREDISPOSITION AND 2480 01:41:45,184 --> 01:41:46,185 THINGS LIKE THAT? 2481 01:41:46,185 --> 01:41:48,020 >> WE KNOW THAT IF WE CAN GRAB 2482 01:41:48,020 --> 01:41:49,888 SOME THINGS FROM ALLERGY, FROM 2483 01:41:49,888 --> 01:41:52,291 NIAID, WE'LL GET GOOD 2484 01:41:52,291 --> 01:41:53,625 IMMUNOPHENOTYPING. 2485 01:41:53,625 --> 01:41:55,294 BECAUSE THEY'VE ACTUALLY BEEN 2486 01:41:55,294 --> 01:41:56,161 INVOLVED IN HELPING US DEVELOP 2487 01:41:56,161 --> 01:41:57,462 THIS PROGRAM, SO WE'VE MODELED 2488 01:41:57,462 --> 01:42:00,432 IT AND WE'VE MADE SURE THAT THE 2489 01:42:00,432 --> 01:42:03,402 TYPE OF THE TYPE OF PHENOTYPING 2490 01:42:03,402 --> 01:42:04,937 BEING DONE HERE IS CONSISTENT 2491 01:42:04,937 --> 01:42:05,904 WITH WHAT THEY THINK IS 2492 01:42:05,904 --> 01:42:06,705 IMPORTANT AND WHAT THEY'RE DOING 2493 01:42:06,705 --> 01:42:07,673 IN THEIR PROGRAM. 2494 01:42:07,673 --> 01:42:09,741 SO THIS WILL LINE YOU PRETTY 2495 01:42:09,741 --> 01:42:09,942 WELL. 2496 01:42:09,942 --> 01:42:11,743 NOW THERE MAY NOT BE QUITE AS 2497 01:42:11,743 --> 01:42:12,845 MUCH PULMONARY FUNCTION 2498 01:42:12,845 --> 01:42:13,712 MEASUREMENTS OR OTHER THINGS WE 2499 01:42:13,712 --> 01:42:14,913 MIGHT DO WITH THESE KIDS THAT 2500 01:42:14,913 --> 01:42:17,115 THEY'RE NOT DOING, BUT WE'LL TRY 2501 01:42:17,115 --> 01:42:18,517 TO GET THE ALIGNMENT AS BEST WE 2502 01:42:18,517 --> 01:42:18,984 CAN. 2503 01:42:18,984 --> 01:42:21,053 IT'S NEVER GOING TO BE TOTALLY 2504 01:42:21,053 --> 01:42:22,054 HARMONIZED OR PERFECT, GIVEN 2505 01:42:22,054 --> 01:42:23,589 THAT WE'RE TRYING TO GRAB THEM 2506 01:42:23,589 --> 01:42:25,123 FROM DIFFERENT SOURCES AT 2507 01:42:25,123 --> 01:42:26,625 DIFFERENT TIMES, BUT WE'LL SEE 2508 01:42:26,625 --> 01:42:28,093 WHAT WE CAN DO TO GET THIS 2509 01:42:28,093 --> 01:42:28,327 CORRECT. 2510 01:42:28,327 --> 01:42:31,496 >> AND THE OTHER QUESTION I HAD 2511 01:42:31,496 --> 01:42:34,766 OR COMMENT IS ABOUT NEONATOLOGY 2512 01:42:34,766 --> 01:42:35,767 INPUT, JUST BECAUSE PRETERM 2513 01:42:35,767 --> 01:42:38,704 BIRTH IS SUCH A RISK FACTOR FOR 2514 01:42:38,704 --> 01:42:40,239 ALL OF THIS, AND I DON'T KNOW IF 2515 01:42:40,239 --> 01:42:41,406 THAT'S GOING TO BE JUST PART OF 2516 01:42:41,406 --> 01:42:46,812 THE COHORT OR YOU WILL EXCLUDE, 2517 01:42:46,812 --> 01:42:50,849 OBVIOUSLY BPD IS AN UNDERLYING 2518 01:42:50,849 --> 01:42:51,984 DISEASE AND MAJOR RISK FACTOR 2519 01:42:51,984 --> 01:42:52,751 FOR ANY OF THEM. 2520 01:42:52,751 --> 01:42:56,488 SO JUST TO MAKE SURE THAT THIS 2521 01:42:56,488 --> 01:42:58,690 UNIQUE NEONATAL -- PARTICULARLY 2522 01:42:58,690 --> 01:43:01,560 PREMATURITY WITH OR WITHOUT LUNG 2523 01:43:01,560 --> 01:43:02,861 DISEASE IS CONSIDERED IN THE 2524 01:43:02,861 --> 01:43:03,128 EQUATION. 2525 01:43:03,128 --> 01:43:06,064 IT WOULD BE GREAT TO INCLUDE 2526 01:43:06,064 --> 01:43:07,799 THEM, BUT THEY'RE SEPARATE, 2527 01:43:07,799 --> 01:43:08,300 DIFFERENT SUBTYPES. 2528 01:43:08,300 --> 01:43:11,436 >> WELL, AGAIN, THIS HAS A LOT 2529 01:43:11,436 --> 01:43:13,438 OF BREADTH TO IF YOU WANT TO TRY 2530 01:43:13,438 --> 01:43:15,073 TO CONTINUE TO EXPAND, AND WE'LL 2531 01:43:15,073 --> 01:43:16,375 TRY TO WRAP IN AS MUCH AS WE 2532 01:43:16,375 --> 01:43:17,175 CAN. 2533 01:43:17,175 --> 01:43:17,709 APPRECIATE THESE COMMENTS, 2534 01:43:17,709 --> 01:43:17,943 THOUGH. 2535 01:43:17,943 --> 01:43:19,511 THEY'RE VERY HELPFUL AS WE THINK 2536 01:43:19,511 --> 01:43:21,246 ABOUT HOW TO BEST STRUCTURE THIS 2537 01:43:21,246 --> 01:43:22,414 GOING FORWARD, WHAT WE CAN 2538 01:43:22,414 --> 01:43:23,916 COLLECT, HOW WE CAN DO IT WITHIN 2539 01:43:23,916 --> 01:43:25,984 A PROJECT THAT WE THINK WE CAN 2540 01:43:25,984 --> 01:43:27,185 MAYBE HANDLE IF THIS GOES 2541 01:43:27,185 --> 01:43:27,619 FORWARD. 2542 01:43:27,619 --> 01:43:28,387 SO APPRECIATE THAT. 2543 01:43:28,387 --> 01:43:30,389 IT'S VERY HELPFUL. 2544 01:43:30,389 --> 01:43:31,790 YES. 2545 01:43:31,790 --> 01:43:34,092 OTHER COMMENTS ON VINYLS? 2546 01:43:34,092 --> 01:43:36,328 IF NOT, IF YOU COULD GO IN TO 2547 01:43:36,328 --> 01:43:38,163 THE SYSTEM AND RATE EACH OF 2548 01:43:38,163 --> 01:43:42,100 THESE ELEMENTS. 2549 01:43:42,100 --> 01:43:43,535 BEFORE WE MOVE ON TO THE NEXT 2550 01:43:43,535 --> 01:43:53,612 ONE. 2551 01:44:03,822 --> 01:44:05,590 >> WE HAVE A FEW PEOPLE THAT ARE 2552 01:44:05,590 --> 01:44:06,358 STILL VOTING. 2553 01:44:06,358 --> 01:44:07,025 OR RATING. 2554 01:44:07,025 --> 01:44:10,862 >> GIVE US THE SIGNAL. 2555 01:44:10,862 --> 01:44:11,964 OH, YOU HAVE TO DO EACH ONE OF 2556 01:44:11,964 --> 01:44:12,497 THEM SEPARATELY. 2557 01:44:12,497 --> 01:44:13,598 I'M SORRY, I SHOULD HAVE 2558 01:44:13,598 --> 01:44:14,599 MENTIONED THAT TO BEGIN WITH. 2559 01:44:14,599 --> 01:44:16,201 DO THE FIRST, AND THEN GO TO THE 2560 01:44:16,201 --> 01:44:20,872 SECOND. 2561 01:44:20,872 --> 01:44:24,710 >> THEY'RE GOING TO VOTE FOR 2562 01:44:24,710 --> 01:44:34,953 3938 AND 3940. 2563 01:44:38,790 --> 01:44:40,359 >> MAYBE JUST ANOTHER MINUTE OR 2564 01:44:40,359 --> 01:44:50,669 SO, OR 30 SECONDS. 2565 01:44:56,708 --> 01:44:58,043 OKAY, I THINK YOU CAN MOVE ALONG 2566 01:44:58,043 --> 01:44:58,210 NOW. 2567 01:44:58,210 --> 01:44:58,977 >> OKAY, THANK YOU. 2568 01:44:58,977 --> 01:45:04,349 SO THE NEXT ONE IS CONCEPT 2569 01:45:04,349 --> 01:45:05,984 NUMBER 5019. 2570 01:45:05,984 --> 01:45:07,185 THIS IS A CONTRACT PROPOSAL, AND 2571 01:45:07,185 --> 01:45:12,557 THIS IS THE RENEWAL OF THE 2572 01:45:12,557 --> 01:45:14,292 NATIONAL SLEEP RESEARCH 2573 01:45:14,292 --> 01:45:15,093 RESOURCE, THE NSRR. 2574 01:45:15,093 --> 01:45:16,962 THIS IS A CONTRACT, THIS IS THE 2575 01:45:16,962 --> 01:45:18,597 RENEWAL TO CONTINUE THAT PROGRAM 2576 01:45:18,597 --> 01:45:20,999 AND HERE WE PROPOSE TO EXPAND 2577 01:45:20,999 --> 01:45:25,637 THIS VERY, VERY HIGHLY UTILIZED 2578 01:45:25,637 --> 01:45:27,072 SLEEP-SPECIFIC PHENOTYPE AND 2579 01:45:27,072 --> 01:45:28,507 PHYSIOLOGICAL SIGNAL DATA 2580 01:45:28,507 --> 01:45:31,810 RESOURCE TO FACILITATE PROPER 2581 01:45:31,810 --> 01:45:35,647 INGESTION OF NEW DATA TYPES, 2582 01:45:35,647 --> 01:45:38,183 MOST NOTABLY THE HUMAN CIRCADIAN 2583 01:45:38,183 --> 01:45:39,518 DATA, AS WELL AS THE ANNOTATION 2584 01:45:39,518 --> 01:45:41,586 AND ENRICHMENT OF THE SLEEP AND 2585 01:45:41,586 --> 01:45:45,991 CIRCADIAN MODEL ORGANISM DATA 2586 01:45:45,991 --> 01:45:47,292 THAT WE'D ALSO LIKE TO 2587 01:45:47,292 --> 01:45:48,193 INCORPORATE INTO THIS RESOURCE 2588 01:45:48,193 --> 01:45:49,828 AND MAKE ALL OF THAT USABLE AND 2589 01:45:49,828 --> 01:45:50,729 ACCESSIBLE TO THE BROADER 2590 01:45:50,729 --> 01:45:52,264 AUDIENCE AND COMMUNITY. 2591 01:45:52,264 --> 01:45:54,232 SO JUST AS A LITTLE BACKGROUND, 2592 01:45:54,232 --> 01:45:56,234 THE NSRR WAS STARTED A NUMBER OF 2593 01:45:56,234 --> 01:45:56,668 YEARS AGO. 2594 01:45:56,668 --> 01:46:01,440 IT WAS ACTUALLY A RESOURCE GRANT 2595 01:46:01,440 --> 01:46:03,775 THAT WAS SUBMITTED BY AN 2596 01:46:03,775 --> 01:46:04,910 INVESTIGATOR THAT THEN GOT 2597 01:46:04,910 --> 01:46:06,611 CONVERTED TO A CONTRACT, BUT 2598 01:46:06,611 --> 01:46:08,513 OVER THE YEARS, IT'S REALLY 2599 01:46:08,513 --> 01:46:10,615 GAINED AND EARNED GLOBAL 2600 01:46:10,615 --> 01:46:12,050 RECOGNITION AS THE PREMIERE DATA 2601 01:46:12,050 --> 01:46:13,819 SHARING REPOSITORY FOR THE SLEEP 2602 01:46:13,819 --> 01:46:15,020 RESEARCH COMMUNITY. 2603 01:46:15,020 --> 01:46:17,055 IN FACT, IT'S EVEN EXPANDED 2604 01:46:17,055 --> 01:46:19,124 BEYOND THE SLEEP COMMUNITY NOW. 2605 01:46:19,124 --> 01:46:20,092 IT'S ACCELERATED THE 2606 01:46:20,092 --> 01:46:22,094 FACILITATION OF RELIABLE AND 2607 01:46:22,094 --> 01:46:24,396 WELL ANNOTATED DATA SHARING 2608 01:46:24,396 --> 01:46:25,931 ACROSS MANY WELL CHARACTERIZED 2609 01:46:25,931 --> 01:46:28,233 COHORT STUDIES AND CLINICAL 2610 01:46:28,233 --> 01:46:30,569 TRIALS, BRINGS THEM ALL IN AND 2611 01:46:30,569 --> 01:46:34,139 ADJUSTS FOR THOSE, IT HARMONIZES 2612 01:46:34,139 --> 01:46:35,407 IT THE BEST THEY CAN AND MAKES 2613 01:46:35,407 --> 01:46:36,942 ALL OF THAT DATA AVAILABLE. 2614 01:46:36,942 --> 01:46:39,478 I DON'T HAVE TO TELL THIS GROUP, 2615 01:46:39,478 --> 01:46:41,580 SOME OF THE POLYGRAPHIC DATA IS 2616 01:46:41,580 --> 01:46:42,981 ENORMOUS, HUGE AMOUNTS OF DATA 2617 01:46:42,981 --> 01:46:45,383 CONTAINED IN SOME OF THOSE SLEEP 2618 01:46:45,383 --> 01:46:48,053 STUDIES, SO NSRR HAS DONE A 2619 01:46:48,053 --> 01:46:52,224 GREAT JOB AS AN NIH-WIDE 2620 01:46:52,224 --> 01:46:53,625 RESOURCE, AND IT HAS NOT ONLY 2621 01:46:53,625 --> 01:46:56,628 ALLOWED THAT TO HAPPEN BUT IT IS 2622 01:46:56,628 --> 01:47:00,665 INCORPORATED -- IT HAS 2623 01:47:00,665 --> 01:47:02,300 INCORPORATED NHLBI CLINICAL 2624 01:47:02,300 --> 01:47:05,270 TRIAL COHORTS, IT'S EVEN NOW 2625 01:47:05,270 --> 01:47:07,105 HOUSING INTERNATIONAL ASSETS SO 2626 01:47:07,105 --> 01:47:08,039 IT'S A GLOBAL RESOURCE. 2627 01:47:08,039 --> 01:47:11,109 IT HAS ABOUT 11,000 USERS 2628 01:47:11,109 --> 01:47:13,111 WORLDWIDE, HAS ABOUT 5,000 DATA 2629 01:47:13,111 --> 01:47:13,879 USE AGREEMENTS. 2630 01:47:13,879 --> 01:47:16,414 THERE ARE CURRENTLY 18 FUNDED 2631 01:47:16,414 --> 01:47:19,351 U.S. PROJECTS FROM ACROSS NIH 2632 01:47:19,351 --> 01:47:20,952 THAT FOCUS ON SLEEP AND 2633 01:47:20,952 --> 01:47:23,688 CIRCADIAN RESEARCH AND CLOSE TO 2634 01:47:23,688 --> 01:47:24,556 400 PUBLICATIONS THAT ARE COMING 2635 01:47:24,556 --> 01:47:26,091 OUT OF THIS RESOURCE. 2636 01:47:26,091 --> 01:47:29,261 SO THIS WAS ALSO REVIEWED BY THE 2637 01:47:29,261 --> 01:47:31,163 BOARD OF EXTERNAL EXPERTS. 2638 01:47:31,163 --> 01:47:33,231 THEY ALSO HAD SOME ENTHUSIASM 2639 01:47:33,231 --> 01:47:33,899 FOR THIS. 2640 01:47:33,899 --> 01:47:38,403 THE NEXT FUNDING PERIOD, THE 2641 01:47:38,403 --> 01:47:40,272 NSRR AS I MENTIONED, WE'D LIKE 2642 01:47:40,272 --> 01:47:42,307 TO EXPAND AND BRING IN SOME NEW 2643 01:47:42,307 --> 01:47:43,808 DATASETS, AND I MENTIONED THE 2644 01:47:43,808 --> 01:47:46,211 HUMAN CIRCADIAN DATA AND MODEL 2645 01:47:46,211 --> 01:47:47,679 ORGANISM, SLEEP AND CIRCADIAN 2646 01:47:47,679 --> 01:47:48,880 DATA, AND I THINK THAT GIVEN THE 2647 01:47:48,880 --> 01:47:50,949 SUCCESS THAT THEY'VE HAD THE 2648 01:47:50,949 --> 01:47:52,250 WITH THE HUMAN SLEEP DATA, THIS 2649 01:47:52,250 --> 01:47:54,486 SHOULD BE VERY DOABLE. 2650 01:47:54,486 --> 01:47:56,888 THEY THEN HAVE A PLAN TO 2651 01:47:56,888 --> 01:47:59,324 TRANSFORM ALL OF THAT DATA AND 2652 01:47:59,324 --> 01:48:00,992 THE RELATED FIELDS INTO A 2653 01:48:00,992 --> 01:48:03,495 RESOURCE THAT WILL ULTIMATELY 2654 01:48:03,495 --> 01:48:09,067 LIVE AND RESIDE WITHIN OUR OWN 2655 01:48:09,067 --> 01:48:11,403 DATASET, BDC, HERE AT NHLBI. 2656 01:48:11,403 --> 01:48:13,705 THIS IS ALSO A TIME WHERE I 2657 01:48:13,705 --> 01:48:17,209 THINK THEY CAN ALSO GENERATE AND 2658 01:48:17,209 --> 01:48:19,644 HELP ADVANCE NEW HYPOTHESES 2659 01:48:19,644 --> 01:48:21,413 USING ARTIFICIAL INTELLIGENCE, 2660 01:48:21,413 --> 01:48:24,015 MACHINE LEARNING APPROACHES, AND 2661 01:48:24,015 --> 01:48:25,383 REALLY MINE ALL OF THAT DATA IN 2662 01:48:25,383 --> 01:48:27,686 A MUCH MORE ROBUST AND 2663 01:48:27,686 --> 01:48:28,887 SOPHISTICATED WAY THAN MAYBE 2664 01:48:28,887 --> 01:48:30,822 WHAT'S BEEN DONE HERETOFORE. 2665 01:48:30,822 --> 01:48:31,990 SO THIS WILL OPEN UP THE 2666 01:48:31,990 --> 01:48:33,758 POSSIBILITY OF MAYBE NOVEL 2667 01:48:33,758 --> 01:48:34,960 SCIENTIFIC QUESTIONS IN SLEEP 2668 01:48:34,960 --> 01:48:37,862 AND CIRCADIAN, AND THEN 2669 01:48:37,862 --> 01:48:39,364 HOPEFULLY PROMPT SOME ADDITIONAL 2670 01:48:39,364 --> 01:48:42,234 RESEARCH THAT WOULD BE COMING 2671 01:48:42,234 --> 01:48:45,070 FROM THIS HYPOTHESIS GENERATING 2672 01:48:45,070 --> 01:48:46,104 KINDS OF A RESOURCE. 2673 01:48:46,104 --> 01:48:47,939 IT ALIGNS VERY WELL WITH THE NIH 2674 01:48:47,939 --> 01:48:51,243 SLEEP RESEARCH PLAN, WITH THE 2675 01:48:51,243 --> 01:48:52,811 NHLBI STRATEGIC REFRESH, AND WE 2676 01:48:52,811 --> 01:48:56,281 THINK THIS IS A VALUABLE 2677 01:48:56,281 --> 01:48:57,182 RESOURCE TO CONTINUE SUPPORTING. 2678 01:48:57,182 --> 01:48:58,817 SO WITH THAT, I'LL STOP AND I'LL 2679 01:48:58,817 --> 01:48:59,951 BE HAPPY TO TRY TO ANSWER 2680 01:48:59,951 --> 01:49:01,152 QUESTIONS HERE AS WELL. 2681 01:49:01,152 --> 01:49:02,787 THIS COMES FROM OUR NATIONAL 2682 01:49:02,787 --> 01:49:04,456 CENTER ON SLEEP DISORDERS 2683 01:49:04,456 --> 01:49:08,193 RESEARCH, LED BY MARISHKA BROWN 2684 01:49:08,193 --> 01:49:10,061 AND STAFF IN THE SLEEP CENTER, 2685 01:49:10,061 --> 01:49:11,529 AND THEY'VE DONE A GOOD JOB OF 2686 01:49:11,529 --> 01:49:14,799 REALLY TRYING TO LOOK AT THIS 2687 01:49:14,799 --> 01:49:16,768 LONG TERM TO MAKE SURE THIS IS 2688 01:49:16,768 --> 01:49:19,237 THE BEST THEY CAN BE AND WELL 2689 01:49:19,237 --> 01:49:20,472 UTILIZED BY THE BROADEST 2690 01:49:20,472 --> 01:49:23,174 COMMUNITY AS POSSIBLE. 2691 01:49:23,174 --> 01:49:29,514 OPEN IT UP FOR QUESTIONS. 2692 01:49:29,514 --> 01:49:31,750 GLL HOW M 2693 01:49:31,750 --> 01:49:33,585 >> HOW MUCH PEDIATRIC DATA IS 2694 01:49:33,585 --> 01:49:35,253 THERE OR FOCUS ON PEDIATRIC 2695 01:49:35,253 --> 01:49:35,553 POPULATIONS? 2696 01:49:35,553 --> 01:49:36,721 >> OH, BOY, THAT'S A GOOD 2697 01:49:36,721 --> 01:49:37,255 QUESTION. 2698 01:49:37,255 --> 01:49:38,590 I DON'T KNOW IF I HAVE AN ANSWER 2699 01:49:38,590 --> 01:49:41,459 TO THAT. 2700 01:49:41,459 --> 01:49:42,394 MARISHKA, ARE YOU THERE WITH US? 2701 01:49:42,394 --> 01:49:43,328 >> YES, I AM HERE. 2702 01:49:43,328 --> 01:49:44,863 >> DID YOU HEAR THE QUESTION? 2703 01:49:44,863 --> 01:49:45,597 WE CAN HEAR YOU. 2704 01:49:45,597 --> 01:49:47,732 >> YEAH, SO THANK YOU, THAT'S A 2705 01:49:47,732 --> 01:49:48,300 GREAT QUESTION. 2706 01:49:48,300 --> 01:49:49,934 WE DO HAVE QUITE A BIT OF 2707 01:49:49,934 --> 01:49:52,170 PEDIATRIC DATA, AND WE'RE 2708 01:49:52,170 --> 01:49:54,673 CONTINUING TO ACTUALLY INGEST 2709 01:49:54,673 --> 01:49:56,107 MORE, SO RIGHT NOW I CAN'T TELL 2710 01:49:56,107 --> 01:49:59,644 YOU THE EXACT NUMBER BUT NSRR 2711 01:49:59,644 --> 01:50:00,845 INGESTS DATA FROM ACROSS THE 2712 01:50:00,845 --> 01:50:01,212 LIFESPAN. 2713 01:50:01,212 --> 01:50:06,685 SO PEDIATRIC DATA DOES EXIST AND 2714 01:50:06,685 --> 01:50:09,220 SOME OF THE MOST POPULAR 2715 01:50:09,220 --> 01:50:12,023 PUBLICATIONS THAT HAVE COME OUT 2716 01:50:12,023 --> 01:50:14,459 OF NSRR HAVE COME OUT OF 2717 01:50:14,459 --> 01:50:15,727 PEDIATRICS AND SLEEP DISORDERED 2718 01:50:15,727 --> 01:50:15,994 BREATHING. 2719 01:50:15,994 --> 01:50:16,328 >> THANK YOU. 2720 01:50:16,328 --> 01:50:18,730 >> OTHER QUESTIONS ON THE SLEEP 2721 01:50:18,730 --> 01:50:19,130 RESEARCH RESOURCE? 2722 01:50:19,130 --> 01:50:25,737 THANK YOU, M MAR ISHKA. 2723 01:50:25,737 --> 01:50:27,505 OKAY, AS WITH THE PREVIOUS ONE, 2724 01:50:27,505 --> 01:50:29,374 IF YOU COULD RATE EACH OF THESE 2725 01:50:29,374 --> 01:50:31,142 ELEMENTS. 2726 01:50:31,142 --> 01:50:34,245 THIS IS 5019. 2727 01:50:34,245 --> 01:50:44,622 THERE'S JUST ONE HERE. 2728 01:50:46,591 --> 01:50:52,797 >> DR. KI 2729 01:50:52,797 --> 01:50:53,998 >> THE LAST ONE I'M GOING TO 2730 01:50:53,998 --> 01:50:56,868 PRESENT TO YOU TODAY IS NUMBER 2731 01:50:56,868 --> 01:50:57,068 5403. 2732 01:50:57,068 --> 01:50:59,471 THIS IS ACTUALLY AN NHLBI-WIDE, 2733 01:50:59,471 --> 01:51:00,939 IN FACT ALMOST -- NOW IT'S GOING 2734 01:51:00,939 --> 01:51:03,675 TO BECOME AN NIH-WIDE 2735 01:51:03,675 --> 01:51:07,212 INITIATIVE, AND WE WOULD LIKE TO 2736 01:51:07,212 --> 01:51:09,080 PARTICIPATE AS A SECONDARY 2737 01:51:09,080 --> 01:51:12,050 SIGN-ON WITH THE NATIONAL 2738 01:51:12,050 --> 01:51:13,017 INSTITUTE ON DIABETES AND 2739 01:51:13,017 --> 01:51:15,620 DIGESTIVE AND KIDNEY DISEASES, 2740 01:51:15,620 --> 01:51:19,057 AS THEY REISSUE A PROGRAM CALLED 2741 01:51:19,057 --> 01:51:21,926 STEP-UP, WHICH IS A SHORT-TERM 2742 01:51:21,926 --> 01:51:24,162 RESEARCH EXPERIENCE PROGRAM TO 2743 01:51:24,162 --> 01:51:24,596 UNLOCK POTENTIAL. 2744 01:51:24,596 --> 01:51:27,966 AS I SAID, THIS IS A 2745 01:51:27,966 --> 01:51:30,368 CROSS-INSTITUTE EFFORT WHERE WE 2746 01:51:30,368 --> 01:51:32,737 WANT TO JOIN NIDDK IN THEIR 2747 01:51:32,737 --> 01:51:34,038 PROGRAM THAT THEY'RE GOING TO 2748 01:51:34,038 --> 01:51:34,472 RENEW. 2749 01:51:34,472 --> 01:51:35,807 SO THIS IS A FIRST-TIME 2750 01:51:35,807 --> 01:51:37,809 PARTICIPATION FOR US. 2751 01:51:37,809 --> 01:51:39,577 THIS IS A -- THE STEP-UP PROGRAM 2752 01:51:39,577 --> 01:51:42,280 IS REALLY TARGETING HIGH 2753 01:51:42,280 --> 01:51:45,450 SCHOOL-LEVEL INDIVIDUALS THAT 2754 01:51:45,450 --> 01:51:46,584 WE'RE TRYING TO ATTRACT INTO 2755 01:51:46,584 --> 01:51:47,652 RESEARCH CAREERS. 2756 01:51:47,652 --> 01:51:49,788 IT WAS FORMALLY ESTABLISHED IN 2757 01:51:49,788 --> 01:51:54,492 2002, AS A RESEARCH EDUCATION 2758 01:51:54,492 --> 01:51:56,261 PROGRAM TO FUND A COORDINATING 2759 01:51:56,261 --> 01:51:59,097 CENTER THAT WOULD ORGANIZE THE 2760 01:51:59,097 --> 01:52:00,765 RECRUITMENT MENTORING AND 2761 01:52:00,765 --> 01:52:02,167 HANDS-ON RESEARCH EXPERIENCE FOR 2762 01:52:02,167 --> 01:52:04,202 HIGH SCHOOL AND PERHAPS 2763 01:52:04,202 --> 01:52:05,937 UNDERGRADUATE STUDENTS IN 2764 01:52:05,937 --> 01:52:07,038 DIVERSE GEOGRAPHIC LOCATIONS 2765 01:52:07,038 --> 01:52:09,707 ACROSS THE U.S. -- ACROSS THE 2766 01:52:09,707 --> 01:52:10,275 UNITED STATES AND U.S. 2767 01:52:10,275 --> 01:52:14,212 TERRITORIES. 2768 01:52:14,212 --> 01:52:16,281 ONE OF ITS GOALS IS TO GROW A 2769 01:52:16,281 --> 01:52:20,685 DIVERSE BIOMEDICAL RESEARCH 2770 01:52:20,685 --> 01:52:23,321 WORKFORCE THAT WOULD HELP 2771 01:52:23,321 --> 01:52:24,489 ADVANCE STUDENTS TO ADDITIONAL 2772 01:52:24,489 --> 01:52:28,359 CAREER LEVELS IN SCIENCE. 2773 01:52:28,359 --> 01:52:29,727 THIS IS A CRITICAL NEED, I DON'T 2774 01:52:29,727 --> 01:52:30,829 HAVE TO SPEAK TO THIS GROUP 2775 01:52:30,829 --> 01:52:33,665 ABOUT THAT. 2776 01:52:33,665 --> 01:52:34,866 IN FACT, WE'RE VERY EXCITED 2777 01:52:34,866 --> 01:52:35,900 ABOUT THIS BECAUSE IT'S ONE OF 2778 01:52:35,900 --> 01:52:37,535 THE FIRST TIMES THAT WE IN THIS 2779 01:52:37,535 --> 01:52:38,636 INSTITUTE ARE REALLY BEGINNING 2780 01:52:38,636 --> 01:52:41,039 TO NOW MEANINGFULLY ENGAGE WITH 2781 01:52:41,039 --> 01:52:46,010 HIGH SCHOOL STUDENTS FROM 2782 01:52:46,010 --> 01:52:46,978 DIVERSE BACKGROUNDS IN 2783 01:52:46,978 --> 01:52:47,979 GEOGRAPHIC LOCATIONS WHO 2784 01:52:47,979 --> 01:52:50,048 HISTORICALLY HAVE INADEQUATE 2785 01:52:50,048 --> 01:52:52,050 ACCESS TO SIGNIFICANT SCIENTIFIC 2786 01:52:52,050 --> 01:52:52,951 RESEARCH OPPORTUNITIES AND WE 2787 01:52:52,951 --> 01:52:54,486 THINK THIS STEP-UP PROGRAM IS 2788 01:52:54,486 --> 01:52:55,687 DESIGNED TO ADDRESS A BARRIER 2789 01:52:55,687 --> 01:52:57,689 AND A CHALLENGE THAT IS FACED BY 2790 01:52:57,689 --> 01:52:59,757 HIGH SCHOOL STUDENTS, AND THIS 2791 01:52:59,757 --> 01:53:02,727 PROGRAM THEN GIVES IT A BOOST TO 2792 01:53:02,727 --> 01:53:05,196 TRY TO ATTRACT, RETAIN AND THEN 2793 01:53:05,196 --> 01:53:07,499 HOPEFULLY ADVANCE THOSE STUDENTS 2794 01:53:07,499 --> 01:53:08,700 THROUGH TYPICAL CAREERS IN 2795 01:53:08,700 --> 01:53:10,335 SCIENCE. 2796 01:53:10,335 --> 01:53:11,603 IT'S NOVEL BECAUSE THE 2797 01:53:11,603 --> 01:53:13,338 COORDINATING CENTERS ARE 2798 01:53:13,338 --> 01:53:16,074 DISTRIBUTED GEOGRAPHICALLY 2799 01:53:16,074 --> 01:53:18,009 ACROSS THE COUNTRY, SO THEY 2800 01:53:18,009 --> 01:53:19,511 FACILITATE SORT OF A STREAMLINED 2801 01:53:19,511 --> 01:53:21,179 SUBMISSION, THEY REVIEW THE 2802 01:53:21,179 --> 01:53:21,813 APPLICATIONS IN HOUSE, THEY 2803 01:53:21,813 --> 01:53:23,648 MATCH THE MENTORS AND THE 2804 01:53:23,648 --> 01:53:27,085 STUDENTS, AND THEN THEY ENSURE 2805 01:53:27,085 --> 01:53:29,120 TIMELY RESEARCH INTERNSHIP. 2806 01:53:29,120 --> 01:53:31,122 RIGHT NOW THE HIGH SCHOOL 2807 01:53:31,122 --> 01:53:33,424 COORDINATING CENTERS SUPPORT 25 2808 01:53:33,424 --> 01:53:34,959 PARTICIPANTS ANNUALLY, AND THE 2809 01:53:34,959 --> 01:53:37,362 STUDENTS ENGAGE IN HANDS-ON 2810 01:53:37,362 --> 01:53:39,731 EXPERIENCE IN THEIR COMMUNITIES, 2811 01:53:39,731 --> 01:53:41,232 MOSTLY WITHIN COMMUTING DISTANCE 2812 01:53:41,232 --> 01:53:42,634 OF THEIR HOMES FOR EIGHT WEEKS 2813 01:53:42,634 --> 01:53:43,101 IN THE SUMMER. 2814 01:53:43,101 --> 01:53:46,237 AT THE END OF THE SUMMER, THEY 2815 01:53:46,237 --> 01:53:48,106 COME BACK AND -- SORRY -- THEY 2816 01:53:48,106 --> 01:53:50,074 ATTEND AN ANNUAL SYMPOSIUM AT 2817 01:53:50,074 --> 01:53:51,976 NIH HERE ON THE CAMPUS, WHERE 2818 01:53:51,976 --> 01:53:55,146 THEY PRESENT THEIR PROJECTS AND 2819 01:53:55,146 --> 01:53:56,347 THEY INTERACT WITH EACH OTHER. 2820 01:53:56,347 --> 01:54:00,218 SO WE THINK THIS IS A MODEST 2821 01:54:00,218 --> 01:54:03,087 INVESTMENT TO ADD ADDITIONAL 2822 01:54:03,087 --> 01:54:06,891 COORDINATING CENTERS FROM NHLBI 2823 01:54:06,891 --> 01:54:09,160 TO THE NIDDY COLLECTION THAT 2824 01:54:09,160 --> 01:54:10,562 WOULD THEN ALLOW US TO ALSO 2825 01:54:10,562 --> 01:54:11,763 TARGET THESE STUDENTS AND 2826 01:54:11,763 --> 01:54:15,800 HOPEFULLY CONVINCE THEM THAT 2827 01:54:15,800 --> 01:54:16,568 CARDIOVASCULAR, PULMONARY, 2828 01:54:16,568 --> 01:54:17,769 HEMATOLOGY AND SLEEP ARE ALSO 2829 01:54:17,769 --> 01:54:20,638 GOOD CAREER OPPORTUNITIES FOR 2830 01:54:20,638 --> 01:54:22,373 THEM TO CONSIDER AS THEY ADVANCE 2831 01:54:22,373 --> 01:54:23,007 IN SCIENCE. 2832 01:54:23,007 --> 01:54:24,108 SO I WILL STOP THERE AND ANSWER 2833 01:54:24,108 --> 01:54:32,283 QUESTIONS ON THIS ONE. 2834 01:54:32,283 --> 01:54:33,785 SORRY, WAS THERE A QUESTION? 2835 01:54:33,785 --> 01:54:34,752 YES, TINA. 2836 01:54:34,752 --> 01:54:35,954 YOU TAKE THE PRIZE HERE SO FAR. 2837 01:54:35,954 --> 01:54:37,388 >> I REALLY LIKE THIS. 2838 01:54:37,388 --> 01:54:39,424 I THINK IT'S IMPORTANT, 2839 01:54:39,424 --> 01:54:40,158 HOPEFULLY IMPACTFUL. 2840 01:54:40,158 --> 01:54:41,459 ONE IDEA THAT I'D LOVE TO SEE 2841 01:54:41,459 --> 01:54:43,094 ADDED TO THIS IS WE'RE TRYING TO 2842 01:54:43,094 --> 01:54:45,129 FIGURE OUT HOW DO THIS ON OUR 2843 01:54:45,129 --> 01:54:48,967 OWN BECAUSE THERE'S SO MANY 2844 01:54:48,967 --> 01:54:50,802 NIH-FUNDED STUDIES THAT ALREADY 2845 01:54:50,802 --> 01:54:51,669 ENGAGE THIS AGE POPULATION. 2846 01:54:51,669 --> 01:54:52,637 ONE OF THE THINGS WE'RE TRYING 2847 01:54:52,637 --> 01:54:54,806 TO DO IS MAKE OUR RESEARCH 2848 01:54:54,806 --> 01:54:56,341 PARTICIPANTS FEEL LIKE CITIZEN 2849 01:54:56,341 --> 01:54:59,010 SIGNISCIENTISTS SO WE'RE KIND OF 2850 01:54:59,010 --> 01:55:00,645 DOING THIS ON OUR OWN. 2851 01:55:00,645 --> 01:55:04,782 I JUST REALIZED THE AMOUNT OF 2852 01:55:04,782 --> 01:55:06,317 RESEARCH POTENTIAL -- THAT WE 2853 01:55:06,317 --> 01:55:07,952 COULD ALSO REACH WITH AN 2854 01:55:07,952 --> 01:55:09,420 INITIATIVE LIKE THIS TO HELP 2855 01:55:09,420 --> 01:55:09,988 INVESTIGATORS, THOSE OF US 2856 01:55:09,988 --> 01:55:11,689 TRYING TO FIGURE OUT ON OUR OWN 2857 01:55:11,689 --> 01:55:13,157 HOW TO MAKE OUR RESEARCH 2858 01:55:13,157 --> 01:55:14,959 PARTICIPANTS NOT FEEL JUST LIKE 2859 01:55:14,959 --> 01:55:16,260 RESEARCH PARTICIPANTS BUT 2860 01:55:16,260 --> 01:55:17,395 CITIZEN SCIENTISTS WOULD BE 2861 01:55:17,395 --> 01:55:18,796 SOMETHING REALLY NICE TO ADD TO 2862 01:55:18,796 --> 01:55:19,097 THIS. 2863 01:55:19,097 --> 01:55:19,564 >> CAN DO. 2864 01:55:19,564 --> 01:55:21,933 I THINK THAT'S A GREAT POINT. 2865 01:55:21,933 --> 01:55:26,070 I'M SURE -- WE'LL WORK WITH D.K. 2866 01:55:26,070 --> 01:55:27,538 ON THIS SINCE THEY'RE DRIVING 2867 01:55:27,538 --> 01:55:28,740 WITH BUT I THINK THEY WOULD 2868 01:55:28,740 --> 01:55:29,507 ABSOLUTELY CONCUR WITH YOUR 2869 01:55:29,507 --> 01:55:32,777 POINTS. 2870 01:55:32,777 --> 01:55:33,344 OKAY. 2871 01:55:33,344 --> 01:55:33,544 LYNN? 2872 01:55:33,544 --> 01:55:35,480 >> I ALSO REALLY LIKE THIS 2873 01:55:35,480 --> 01:55:38,116 PROGRAM AND THE CONCEPT AND THE 2874 01:55:38,116 --> 01:55:40,652 FACT THAT IT'S BEEN AROUND FOR A 2875 01:55:40,652 --> 01:55:42,520 NUMBER OF YEARS AND THERE IS 2876 01:55:42,520 --> 01:55:42,987 METRICS OF SUCCESS. 2877 01:55:42,987 --> 01:55:44,622 MY QUESTION IS, HOW LONG ARE 2878 01:55:44,622 --> 01:55:46,824 THOSE PARTICIPANTS FOLLOWED 2879 01:55:46,824 --> 01:55:48,793 AFTERWARDS, AND ARE THERE 2880 01:55:48,793 --> 01:55:50,061 ANOTHER MECHANISM AFTER GOING 2881 01:55:50,061 --> 01:55:55,933 THROUGH THIS PROGRAM TO CAPTURE 2882 01:55:55,933 --> 01:55:57,468 THOSE INDIVIDUALS AND CONTINUE 2883 01:55:57,468 --> 01:55:58,703 TO FOSTER THEIR INTEREST IN 2884 01:55:58,703 --> 01:55:58,936 SCIENCE? 2885 01:55:58,936 --> 01:56:02,607 >> I THINK THAT CAME UP WITH THE 2886 01:56:02,607 --> 01:56:03,708 B2 AS WELL. 2887 01:56:03,708 --> 01:56:04,776 CAN YOU HELP WITH THAT ONE? 2888 01:56:04,776 --> 01:56:05,777 BECAUSE YOU PROBABLY KNOW THE 2889 01:56:05,777 --> 01:56:15,853 ANSWER. 2890 01:56:15,853 --> 01:56:26,064 [INAUDIBLE] 2891 01:56:43,748 --> 01:56:45,183 >> BECAUSE ALL OF US ARE ALSO 2892 01:56:45,183 --> 01:56:47,485 TRYING TO INCREASE ENROLLMENT IN 2893 01:56:47,485 --> 01:56:50,988 MEDICAL SCHOOL OR IN OUR T -- ET 2894 01:56:50,988 --> 01:56:54,559 CETERA, SO WE CAN I'D -- IF WE 2895 01:56:54,559 --> 01:56:55,760 CAN IDENTIFY A COHORT TO 2896 01:56:55,760 --> 01:56:57,361 CONTINUE TO TAP INTO WOULD BE 2897 01:56:57,361 --> 01:56:59,697 AWESOME. 2898 01:56:59,697 --> 01:57:00,965 >> GOOD POINT TOO. 2899 01:57:00,965 --> 01:57:02,433 >> DOES THE MECHANISM PROVIDE 2900 01:57:02,433 --> 01:57:03,768 ANY SUPPORT FOR THE MENTORS WHO 2901 01:57:03,768 --> 01:57:06,771 ARE HOSTING THEM? 2902 01:57:06,771 --> 01:57:09,140 >> I DO NOT THINK SO. 2903 01:57:09,140 --> 01:57:11,442 DO YOU KNOW THE ANSWER TO THAT 2904 01:57:11,442 --> 01:57:12,510 ONE, MARISOL? 2905 01:57:12,510 --> 01:57:13,711 THEY DON'T GET A SALARY OR ANY 2906 01:57:13,711 --> 01:57:16,247 KIND OF COMPENSATION, DO THEY? 2907 01:57:16,247 --> 01:57:16,414 NO. 2908 01:57:16,414 --> 01:57:18,282 >> THE ONE CHALLENGE I WOULD SAY 2909 01:57:18,282 --> 01:57:20,785 WITH THAT IS, IT'S ONE THING FOR 2910 01:57:20,785 --> 01:57:21,552 RESEARCH ONE INSTITUTIONS THAT 2911 01:57:21,552 --> 01:57:22,987 ARE VERY WELL-FUNDED THAT HAVE 2912 01:57:22,987 --> 01:57:24,655 LAYERS OF PEOPLE WHO ARE ABLE TO 2913 01:57:24,655 --> 01:57:26,624 ENGAGE AND PROVIDE A STRUCTURED 2914 01:57:26,624 --> 01:57:28,359 EXPERIENCE, BUT A LOT OF 2915 01:57:28,359 --> 01:57:28,926 UNDERREPRESENTED INDIVIDUALS 2916 01:57:28,926 --> 01:57:30,428 DON'T LIVE WITHIN A CLOSE 2917 01:57:30,428 --> 01:57:32,263 DISTANCE TO THOSE TYPES OF 2918 01:57:32,263 --> 01:57:33,798 INSTITUTIONS, AND IT CAN BE 2919 01:57:33,798 --> 01:57:35,399 QUITE -- IT CAN TAKE A LOT OF 2920 01:57:35,399 --> 01:57:36,334 TIME, PARTICULARLY FOR STUDENTS 2921 01:57:36,334 --> 01:57:38,236 WHO ARE NOT COMING IN WITH THE 2922 01:57:38,236 --> 01:57:41,706 SAME SKILLSET AS VERY PRIVILEGED 2923 01:57:41,706 --> 01:57:42,373 STUDENTS. 2924 01:57:42,373 --> 01:57:45,209 IT CAN TAKE A LOT MORE TIME TO 2925 01:57:45,209 --> 01:57:47,111 MENTOR THAT MANY MAY NOT BE ABLE 2926 01:57:47,111 --> 01:57:49,413 TO SPARE, AND A LOT OF 2927 01:57:49,413 --> 01:57:51,282 TEACHING-INTENSIVE INSTITUTIONS, 2928 01:57:51,282 --> 01:57:53,251 I UNDERSTAND MAYBE THAT'S NOT 2929 01:57:53,251 --> 01:57:54,418 WHERE THESE ARE BASED BUT THEY 2930 01:57:54,418 --> 01:57:55,720 PROVIDE A GREAT ENTREE INTO 2931 01:57:55,720 --> 01:57:57,054 EDUCATION AND BEING ABLE TO 2932 01:57:57,054 --> 01:57:58,489 PROVIDE SUPPORT FOR THEM WHERE 2933 01:57:58,489 --> 01:58:02,026 THEY DON'T HAVE SUMMER SALARY 2934 01:58:02,026 --> 01:58:02,794 COVERAGE OR OTHER THINGS TO 2935 01:58:02,794 --> 01:58:04,428 PROVIDE A COMPREHENSIVE TRAINING 2936 01:58:04,428 --> 01:58:06,130 EXPERIENCE, I THINK WOULD BE 2937 01:58:06,130 --> 01:58:07,765 VERY IMPORTANT AND EXPAND THE 2938 01:58:07,765 --> 01:58:07,999 IMPACT. 2939 01:58:07,999 --> 01:58:08,533 >> ALL RIGHT. 2940 01:58:08,533 --> 01:58:11,035 WELL, THAT'S AN IMPORTANT POINT. 2941 01:58:11,035 --> 01:58:13,104 IT'S ONE THAT WE'LL HAVE TO TALK 2942 01:58:13,104 --> 01:58:14,505 TO D.K. ABOUT, GARY. 2943 01:58:14,505 --> 01:58:17,341 >> IT IS AN EXCELLENT POINT, AND 2944 01:58:17,341 --> 01:58:20,545 IT MIGHT EVEN HAVE COME UP IN IF 2945 01:58:20,545 --> 01:58:23,648 IN THE SENSE THAT THERE'S 2946 01:58:23,648 --> 01:58:24,882 CLEARLY A GOOD TRACK RECORD, BUT 2947 01:58:24,882 --> 01:58:27,218 THEY PROBABLY COULD USE A 2948 01:58:27,218 --> 01:58:28,753 BROADER DIVERSITY OF THOSE HUBS, 2949 01:58:28,753 --> 01:58:30,188 AND SO ACTUALLY THAT WAS ONE OF 2950 01:58:30,188 --> 01:58:33,124 THE THINGS ABOUT US JOINING IN. 2951 01:58:33,124 --> 01:58:35,493 THEY BUILD UP THE CRITICAL MASS 2952 01:58:35,493 --> 01:58:38,095 OF ICs THAT WOULD ENABLE TO 2953 01:58:38,095 --> 01:58:40,398 SEE AND THEN AS YOU SAY, HAVE 2954 01:58:40,398 --> 01:58:43,501 GREATER OUTREACH. 2955 01:58:43,501 --> 01:58:44,402 SO GREAT POINT AND SOMETHING WE 2956 01:58:44,402 --> 01:58:50,341 CAN WORK WITH D.K. ON. 2957 01:58:50,341 --> 01:58:52,276 >> ANY OTHER COMMENTS ON THIS 2958 01:58:52,276 --> 01:58:52,677 ONE? 2959 01:58:52,677 --> 01:58:54,645 IF NOT, 5043, YOU'RE RATING 2960 01:58:54,645 --> 01:59:01,419 THOSE ELEMENTS AS WELL. 2961 01:59:01,419 --> 01:59:03,221 WHERE DO WE GO NEXT? 2962 01:59:03,221 --> 01:59:03,588 CARDIOVASCULAR? 2963 01:59:03,588 --> 01:59:04,689 >> CARDIOVASCULAR. 2964 01:59:04,689 --> 01:59:06,090 >> WITH THAT, I'M GOING TO TURN 2965 01:59:06,090 --> 01:59:07,592 IT OVER TO DR. GOFF. 2966 01:59:07,592 --> 01:59:10,094 I THINK THEY'RE NEXT, DAVID, 2967 01:59:10,094 --> 01:59:15,132 CARDIOVASCULAR DIVISION? 2968 01:59:15,132 --> 01:59:25,176 >> ARE YOU SURE YOU'RE THROUGH? 2969 01:59:25,176 --> 01:59:25,509 OKAY. 2970 01:59:25,509 --> 01:59:30,915 SO WE HAVE FIVE CONCEPTS TO 2971 01:59:30,915 --> 01:59:34,185 PRESENT FROM THE TI VIS DIVISIOF 2972 01:59:34,185 --> 01:59:35,019 CARDIOVASCULAR SCIENCES TODAY. 2973 01:59:35,019 --> 01:59:37,989 WE'RE STARTING I BELIEVE WITH 2974 01:59:37,989 --> 01:59:38,756 CONCEPT 5027? 2975 01:59:38,756 --> 01:59:39,423 YES. 2976 01:59:39,423 --> 01:59:40,791 THAT'S ON THE SCREEN. 2977 01:59:40,791 --> 01:59:46,797 IT'S THE BENCH TO -- CONGENITAL 2978 01:59:46,797 --> 01:59:55,873 HEART DISEASE ADVANCING NEW 2979 01:59:55,873 --> 01:59:57,708 UNDERSTANDING IN GENOMICS 2980 01:59:57,708 --> 02:00:02,747 COHORT, B2B CHANGE COHORT. 2981 02:00:02,747 --> 02:00:04,482 IT'S A RENEWAL OF AN EXISTING 2982 02:00:04,482 --> 02:00:05,850 INITIATIVE BUT IT'S REALLY A 2983 02:00:05,850 --> 02:00:08,152 TRANSFORMATION OF AN EXISTING 2984 02:00:08,152 --> 02:00:08,920 INITIATIVE AND IT WOULD BE 2985 02:00:08,920 --> 02:00:10,788 FUNDED THROUGH A U01 MECHANISM 2986 02:00:10,788 --> 02:00:15,559 IF WE GO FORWARD. 2987 02:00:15,559 --> 02:00:19,163 IT'S A TRANSFORMATION OF 2988 02:00:19,163 --> 02:00:22,233 PEDIATRICS -- REFERRED TO AS THE 2989 02:00:22,233 --> 02:00:24,702 PCGC THAT'S HAD 15 YEARS OF 2990 02:00:24,702 --> 02:00:26,637 SUPPORT FROM NHLBI. 2991 02:00:26,637 --> 02:00:27,772 IT'S REALLY CHANGED THE 2992 02:00:27,772 --> 02:00:29,407 LANDSCAPE OF WHAT WE KNOW ABOUT 2993 02:00:29,407 --> 02:00:30,207 CONGENITAL HEART DISEASE 2994 02:00:30,207 --> 02:00:30,775 GENETICS. 2995 02:00:30,775 --> 02:00:35,046 WE NOW UNDERSTAND AN ADDITIONAL 2996 02:00:35,046 --> 02:00:37,648 25% OF WHAT WAS PREVIOUSLY 2997 02:00:37,648 --> 02:00:39,283 UNEXPLAINED IN TERMS OF JEK 2998 02:00:39,283 --> 02:00:48,859 DETERMINANGENETICDETERMINANTS OS 2999 02:00:48,859 --> 02:00:51,262 PRIMARILY NEWBORNS TO 35 YEARS 3000 02:00:51,262 --> 02:00:52,863 OLD AND OVER 18,000 RELATIVES, 3001 02:00:52,863 --> 02:00:54,532 MAKING IT ONE OF THE LARGEST 3002 02:00:54,532 --> 02:00:57,368 CONGENITAL HEART DISEASE COHORTS 3003 02:00:57,368 --> 02:00:58,803 WITH GENOMIC AND CLINICAL DATA 3004 02:00:58,803 --> 02:01:00,638 EVER ASSEMBLED. 3005 02:01:00,638 --> 02:01:03,107 SO WE PROPOSE TO LEVERAGE THESE 3006 02:01:03,107 --> 02:01:06,410 PREVIOUS INVESTMENTS TO 3007 02:01:06,410 --> 02:01:08,245 TRANSFORM THE PCGC INTO WHAT 3008 02:01:08,245 --> 02:01:11,148 WE'RE CALLING THE B2B CHANGE 3009 02:01:11,148 --> 02:01:11,382 COHORT. 3010 02:01:11,382 --> 02:01:12,683 THE COHORT WOULD ENHANCE THE 3011 02:01:12,683 --> 02:01:13,985 VALUE OF OUR EXISTING 3012 02:01:13,985 --> 02:01:15,686 INVESTMENTS THROUGH LONG-TERM 3013 02:01:15,686 --> 02:01:18,456 FOLLOW-UP OF ABOUT 6,000 CURRENT 3014 02:01:18,456 --> 02:01:21,292 PCGC PARTICIPANTS, AND ALLOWING 3015 02:01:21,292 --> 02:01:23,961 US TO ASSESS MANY CLINICAL 3016 02:01:23,961 --> 02:01:24,762 OUTCOMES OF INTEREST SUCH AS 3017 02:01:24,762 --> 02:01:26,630 HEART FAILURE AND ARRHYTHMIAS 3018 02:01:26,630 --> 02:01:31,135 THAT HAVE NOT YET OCCURRED BUT 3019 02:01:31,135 --> 02:01:32,236 WILL LIKELY OCCUR IN COHORT 3020 02:01:32,236 --> 02:01:33,771 MEMBERS, MANY OF WHOM AS I 3021 02:01:33,771 --> 02:01:35,206 MENTIONED WERE ENROLLED AS 3022 02:01:35,206 --> 02:01:35,439 INFANTS. 3023 02:01:35,439 --> 02:01:38,209 NOW THE COHORT WILL ALLOW A 3024 02:01:38,209 --> 02:01:40,044 UNIQUE OPPORTUNITY TO UNDERSTAND 3025 02:01:40,044 --> 02:01:40,945 LONG TERM CLINICAL OUTCOMES AND 3026 02:01:40,945 --> 02:01:42,913 HOW THEY RELATE TO A PERSON'S 3027 02:01:42,913 --> 02:01:43,881 UNDERLYING GENETICS. 3028 02:01:43,881 --> 02:01:45,516 IT WILL ALSO PROVIDE A PLATFORM 3029 02:01:45,516 --> 02:01:46,784 FOR MECHANISTIC STUDIES TO 3030 02:01:46,784 --> 02:01:49,754 ADVANCE THE CONGENITAL HEART 3031 02:01:49,754 --> 02:01:52,490 DISEASE FIELD TOWARD THE 3032 02:01:52,490 --> 02:01:53,958 DEVELOPMENT OF PRECISION BASED 3033 02:01:53,958 --> 02:01:55,092 CLINICAL TRIALS AND TARGETED 3034 02:01:55,092 --> 02:01:55,426 THERAPEUTICS. 3035 02:01:55,426 --> 02:01:56,394 SO WITH THE RECENT RENEWAL OF 3036 02:01:56,394 --> 02:01:57,628 THE PEDIATRIC HEART NETWORK, 3037 02:01:57,628 --> 02:02:00,064 WHICH IS KIND OF A COMPLEMENTARY 3038 02:02:00,064 --> 02:02:01,432 EFFORT OF THE INSTITUTE, THERE 3039 02:02:01,432 --> 02:02:02,900 WILL BE AN OPPORTUNITY FOR 3040 02:02:02,900 --> 02:02:04,602 CONTINUED COLLABORATION ACROSS 3041 02:02:04,602 --> 02:02:06,370 THE TRANSLATIONAL SPECTRUM. 3042 02:02:06,370 --> 02:02:09,206 SO THIS INITIATIVE WOULD SUPPORT 3043 02:02:09,206 --> 02:02:11,075 A COHORT INCLUDING COLLECTING 3044 02:02:11,075 --> 02:02:13,744 DATA ON LONGITUDAL CLINICAL 3045 02:02:13,744 --> 02:02:15,513 OUTCOMES, COMMUNITY ENGAGEMENT 3046 02:02:15,513 --> 02:02:17,715 TO OBTAIN A REPRESENTATIVE 3047 02:02:17,715 --> 02:02:21,852 COHORT, IMPORTANT PHENOTYPIC AND 3048 02:02:21,852 --> 02:02:23,120 SOCIAL DETERMINANTS DATA, 3049 02:02:23,120 --> 02:02:26,090 LEVERAGING PHENOTYPIC DATA IN 3050 02:02:26,090 --> 02:02:27,591 RELEVANT CONGENITAL HEART 3051 02:02:27,591 --> 02:02:29,126 DISEASE IN PEDIATRIC REGISTRIES 3052 02:02:29,126 --> 02:02:31,062 AND WOULD FACILITATE AN 3053 02:02:31,062 --> 02:02:32,763 IN-PERSON VISIT DURING YEAR FOUR 3054 02:02:32,763 --> 02:02:34,331 OR FIVE TO SUPPORT ANCILLARY 3055 02:02:34,331 --> 02:02:35,433 STUDIES THAT COULD BE FUNDED 3056 02:02:35,433 --> 02:02:38,702 THROUGH INVESTIGATOR-INITIATED 3057 02:02:38,702 --> 02:02:40,871 GRANT APPLICATIONS, R01, SIMILAR 3058 02:02:40,871 --> 02:02:42,406 TO OUR CONTRACT PLATFORM 3059 02:02:42,406 --> 02:02:43,040 COHORTS. 3060 02:02:43,040 --> 02:02:44,175 ALTHOUGH THIS WOULD BE SUPPORTED 3061 02:02:44,175 --> 02:02:46,310 THROUGH A COOPERATIVE AGREEMENT. 3062 02:02:46,310 --> 02:02:48,712 THE INITIATIVE PROPOSES A BUDGET 3063 02:02:48,712 --> 02:02:50,748 OF ABOUT 5 1/2 MILLION PER YEAR 3064 02:02:50,748 --> 02:02:54,018 FOR SEVEN YEARS, WHICH IS ABOUT 3065 02:02:54,018 --> 02:02:57,088 20% REDUCTION FROM THE CURRENT 3066 02:02:57,088 --> 02:02:57,621 PCGC BUDGET. 3067 02:02:57,621 --> 02:03:00,458 WE THINK THIS IS A REALLY GREAT 3068 02:03:00,458 --> 02:03:01,759 WAY TO LEVERAGE THE INVESTMENTS 3069 02:03:01,759 --> 02:03:03,294 WE'VE MADE TO DATE, AND LOOK 3070 02:03:03,294 --> 02:03:04,495 FORWARD TO YOUR QUESTIONS AND 3071 02:03:04,495 --> 02:03:14,338 COMMENTS. 3072 02:03:14,338 --> 02:03:16,941 >> YOU WERE VERY CLEAR. 3073 02:03:16,941 --> 02:03:20,311 >> APPARENTLY. 3074 02:03:20,311 --> 02:03:22,813 WONDERFUL! 3075 02:03:22,813 --> 02:03:25,616 WELL THEN -- EVERY NOW AND THEN, 3076 02:03:25,616 --> 02:03:25,816 RIGHT? 3077 02:03:25,816 --> 02:03:26,817 WHAT DO THEY SAY ABOUT SQUIRRELS 3078 02:03:26,817 --> 02:03:27,218 AND NUTS? 3079 02:03:27,218 --> 02:03:33,324 PLEASE GO AHEAD AND RATE THIS 3080 02:03:33,324 --> 02:03:37,461 ONE AND WE'LL MOVE ON. 3081 02:03:37,461 --> 02:03:38,529 YOU LET ME KNOW WHEN IT'S TIME 3082 02:03:38,529 --> 02:03:38,896 TO MOVE ON. 3083 02:03:38,896 --> 02:03:39,630 >> OKAY. 3084 02:03:39,630 --> 02:03:41,565 LOOKS LIKE EVERYONE IS STILL 3085 02:03:41,565 --> 02:03:51,876 VOTING, OR RATING. 3086 02:04:22,439 --> 02:04:23,407 THANK YOU, YOU CAN MOVE ON. 3087 02:04:23,407 --> 02:04:27,578 >> NEXT CONCEPT IS 5024, THE 3088 02:04:27,578 --> 02:04:30,114 GLOABL CARDIOVASCULAR FORUM, 3089 02:04:30,114 --> 02:04:32,383 WOMEN'S CARDIOVASCULAR HEALTH 3090 02:04:32,383 --> 02:04:34,051 INITIATIVE. 3091 02:04:34,051 --> 02:04:35,819 THIS IS A INITIATIVE THAT WOULD 3092 02:04:35,819 --> 02:04:38,822 BE FUNDED THROUGH SORT OF AN 3093 02:04:38,822 --> 02:04:41,458 OTHER OR M.O.U. TYPE OF FUNDING 3094 02:04:41,458 --> 02:04:41,992 ARRANGEMENT. 3095 02:04:41,992 --> 02:04:45,029 SO THE GCRFF, IF YOU'LL LET ME 3096 02:04:45,029 --> 02:04:47,665 USE THAT ACRONYM, THE GLOBAL 3097 02:04:47,665 --> 02:04:49,266 CARDIOVASCULAR RESEARCH FUNDERS 3098 02:04:49,266 --> 02:04:51,101 FORUM, IS A PARTNERSHIP MADE UP 3099 02:04:51,101 --> 02:04:52,736 OF 12 MAJOR CARDIOVASCULAR 3100 02:04:52,736 --> 02:04:54,438 RESEARCH FUNDERS INCLUDING 3101 02:04:54,438 --> 02:04:55,439 NHLBI, THE AMERICAN HEART 3102 02:04:55,439 --> 02:04:56,874 ASSOCIATION, AND OTHER FUNDERS 3103 02:04:56,874 --> 02:05:00,244 ACROSS CANADA, EUROPE, AUSTRALIA 3104 02:05:00,244 --> 02:05:01,111 AND NEW ZEALAND, THAT WAS 3105 02:05:01,111 --> 02:05:03,414 ESTABLISHED IN 2018 TO SUPPORT 3106 02:05:03,414 --> 02:05:04,715 AND PROMOTE CARDIOVASCULAR 3107 02:05:04,715 --> 02:05:06,850 RESEARCH. 3108 02:05:06,850 --> 02:05:09,153 THIS IS A SIGNIFICANT SCIENTIFIC 3109 02:05:09,153 --> 02:05:12,022 AREA FOR THE GCRFF BECAUSE CBD 3110 02:05:12,022 --> 02:05:17,127 I -- BECAUSECVD IS THE REMAININF 3111 02:05:17,127 --> 02:05:19,463 DEATH IN WOMEN, IT REMAINS UNDER 3112 02:05:19,463 --> 02:05:22,900 DID YOU STUDIED, UNDERDIAGNOSED 3113 02:05:22,900 --> 02:05:27,938 AND UNDERTREATED, AND MANY 3114 02:05:27,938 --> 02:05:29,440 UNDERSTUDIED AREAS FROM A 3115 02:05:29,440 --> 02:05:33,210 LIFESPAN PERSPECTIVE. 3116 02:05:33,210 --> 02:05:34,612 THE FUNDING AND STRUCTURE OF 3117 02:05:34,612 --> 02:05:37,781 THIS IS THAT WE'RE BEING ASKED 3118 02:05:37,781 --> 02:05:40,818 AS A PARTICIPANT IN THE GCRFF 3119 02:05:40,818 --> 02:05:43,988 WITH OTHER FUNDING AGENCIES TO 3120 02:05:43,988 --> 02:05:44,989 CONTRIBUTE $1.9 MILLION, WHICH 3121 02:05:44,989 --> 02:05:48,692 IS THE SAME AS THE OTHER LARGER 3122 02:05:48,692 --> 02:05:50,761 FUNDING ORGANIZATIONS IN THIS 3123 02:05:50,761 --> 02:05:54,265 CONSORTIUM, INCLUDING THE HEART 3124 02:05:54,265 --> 02:05:55,766 ASSOCIATION, THE LE DUKE 3125 02:05:55,766 --> 02:05:57,101 FOUNDATION AND BRITISH HEART 3126 02:05:57,101 --> 02:05:58,736 FOUNDATION. 3127 02:05:58,736 --> 02:05:59,837 SO $1.9 MILLION WOULD BE OUR 3128 02:05:59,837 --> 02:06:01,839 SHARE OF THE TOTAL OF 3129 02:06:01,839 --> 02:06:05,843 $10 MILLION TO FUND THIS INITIAL 3130 02:06:05,843 --> 02:06:08,445 FIVE-YEAR RESEARCH PROJECT ON 3131 02:06:08,445 --> 02:06:11,081 WOMEN'S CARDIOVASCULAR HEL. 3132 02:06:11,081 --> 02:06:11,615 HEALTH. 3133 02:06:11,615 --> 02:06:12,483 THE INTENT OF THE INITIATIVE IS 3134 02:06:12,483 --> 02:06:14,451 TO FUND A COHESIVE RESEARCH 3135 02:06:14,451 --> 02:06:16,320 PROGRAM THAT WOULD TAP INTO 3136 02:06:16,320 --> 02:06:18,355 EXPERTS FROM AROUND THE WORLD 3137 02:06:18,355 --> 02:06:19,690 AND DELIVER THE TYPE OF IMPACT 3138 02:06:19,690 --> 02:06:21,659 THAT NO SINGLE INSTITUTION OR 3139 02:06:21,659 --> 02:06:25,362 COUNTRY CAN DELIVER ON ITS OWN. 3140 02:06:25,362 --> 02:06:26,530 ONE RESEARCH GRANT WILL BE 3141 02:06:26,530 --> 02:06:29,700 AWARDED IN FISCAL YEAR 26, THE 3142 02:06:29,700 --> 02:06:31,235 HIGHEST RATED INTERNATIONAL 3143 02:06:31,235 --> 02:06:33,404 RESEARCH NETWORK APPLICATION. 3144 02:06:33,404 --> 02:06:34,738 ALTHOUGH IT'S BEING REFERRED TO 3145 02:06:34,738 --> 02:06:36,140 AS A NETWORK, IT'S REALLY MORE 3146 02:06:36,140 --> 02:06:38,108 OF WHAT WE WOULD PROBABLY CALL A 3147 02:06:38,108 --> 02:06:39,510 CONSORTIUM. 3148 02:06:39,510 --> 02:06:42,680 FUNDING WILL BE OPEN TO LEAD 3149 02:06:42,680 --> 02:06:46,050 INSTITUTIONS FROM A GCRFF 3150 02:06:46,050 --> 02:06:46,283 COUNTRY. 3151 02:06:46,283 --> 02:06:49,186 IT'S EXPECTED THAT THE AWARD 3152 02:06:49,186 --> 02:06:52,122 WOULD BE MULTIDISCIPLINARY, 3153 02:06:52,122 --> 02:06:52,790 MULTI-INSTITUTIONAL AND 3154 02:06:52,790 --> 02:06:53,791 MULTI-COUNTRY AND STRUCTURE, AND 3155 02:06:53,791 --> 02:06:56,293 THE LEAD INSTITUTION CAN PARTNER 3156 02:06:56,293 --> 02:06:58,662 WITH INVESTIGATORS FROM 3157 02:06:58,662 --> 02:06:59,196 NON-GCRFF COUNTRIES. 3158 02:06:59,196 --> 02:07:00,864 THIS IS AN ASPECT THAT WAS 3159 02:07:00,864 --> 02:07:05,302 REALLY IMPORTANT TO US AS AN 3160 02:07:05,302 --> 02:07:08,138 INSTITUTE TO MAKE SURE THAT THE 3161 02:07:08,138 --> 02:07:11,208 IMPACT OF THIS RESEARCH WOULD GO 3162 02:07:11,208 --> 02:07:12,176 BEYOND HIGH-INCOME COUNTRIES AND 3163 02:07:12,176 --> 02:07:13,811 REALLY ADDRESS ISSUES OF 3164 02:07:13,811 --> 02:07:15,446 IMPORTANCE TO WOMEN'S 3165 02:07:15,446 --> 02:07:17,648 CARDIOVASCULAR HEALTH GLOBALLY, 3166 02:07:17,648 --> 02:07:19,249 WHERE 80% OF CARDIOVASCULAR 3167 02:07:19,249 --> 02:07:20,617 CONDITIONS AND DISEASES OCCUR IN 3168 02:07:20,617 --> 02:07:22,419 LOW AND MIDDLE INCOME COUNTRIES. 3169 02:07:22,419 --> 02:07:24,021 SO SOME OF THE POTENTIAL 3170 02:07:24,021 --> 02:07:25,989 RESEARCH TOPICS EXAMPLES INCLUDE 3171 02:07:25,989 --> 02:07:26,957 ADVANCING EFFECTIVE PREVENTION 3172 02:07:26,957 --> 02:07:31,095 AND TREATMENT OF CVD IN WOMEN 3173 02:07:31,095 --> 02:07:32,196 ACROSS DIFFERENT COUNTRIES AND 3174 02:07:32,196 --> 02:07:33,530 SOCIAL SETTINGS, UNDERSTANDING 3175 02:07:33,530 --> 02:07:35,366 WHY HEART OWE TACK IS OCCURRING 3176 02:07:35,366 --> 02:07:37,101 AT AN INCREASING RATE IN YOUNGER 3177 02:07:37,101 --> 02:07:38,335 WOMEN GLOBALLY, PROVIDING 3178 02:07:38,335 --> 02:07:39,603 INSIGHT ON PATHOLOGY AND 3179 02:07:39,603 --> 02:07:41,872 TREATMENT OF PERIPARTUM 3180 02:07:41,872 --> 02:07:43,941 CARDIOMYOPATHY, WHICH IS MORE 3181 02:07:43,941 --> 02:07:47,511 PREVALENT IN WOMEN OF AFRICAN 3182 02:07:47,511 --> 02:07:50,280 HISTORY AND WOMEN IN SUB-SAHARAN 3183 02:07:50,280 --> 02:07:50,948 AFRICA. 3184 02:07:50,948 --> 02:07:52,583 TESTING STRATEGIES TO ADVANCE 3185 02:07:52,583 --> 02:07:53,851 THE IMPLEMENTATION OF 3186 02:07:53,851 --> 02:07:55,919 EVIDENCE-BASED PRACTICES RELATED 3187 02:07:55,919 --> 02:07:57,588 TO CARDIOVASCULAR DISEASE IN 3188 02:07:57,588 --> 02:08:00,858 WOMEN ACROSS DIFFERENT SECTORS. 3189 02:08:00,858 --> 02:08:03,127 SO WE BELIEVE THE GCRFF IS 3190 02:08:03,127 --> 02:08:04,461 QUALIFIED TO LEAD THIS EFFORT 3191 02:08:04,461 --> 02:08:06,230 BECAUSE THE MEMBERS HAVE LONG 3192 02:08:06,230 --> 02:08:08,232 TRACK RECORDS OF SUPPORTING 3193 02:08:08,232 --> 02:08:09,900 MERITORIOUS RESEARCH AND HAVE 3194 02:08:09,900 --> 02:08:11,535 DEVELOPED A COLLABORATIVE 3195 02:08:11,535 --> 02:08:13,604 CULTURE THROUGH THE 3196 02:08:13,604 --> 02:08:14,238 MULTI-NATIONAL CLINICAL TRIALS 3197 02:08:14,238 --> 02:08:15,639 INITIATIVE, WHICH WAS THE FIRST 3198 02:08:15,639 --> 02:08:19,576 INITIATIVE OF THIS CONSORTIUM. 3199 02:08:19,576 --> 02:08:21,111 THERE'S ALSO A RESEARCH FUNDING 3200 02:08:21,111 --> 02:08:25,449 DATA SHARING INITIATIVE THAT IS 3201 02:08:25,449 --> 02:08:26,016 THE SECOND WORK STREAM. 3202 02:08:26,016 --> 02:08:27,117 THIS WILL BE THE FIRST TIME THAT 3203 02:08:27,117 --> 02:08:28,952 THE MEMBERS WILL ACTUALLY BE 3204 02:08:28,952 --> 02:08:31,989 POOLING RESOURCES TO AWARD AN 3205 02:08:31,989 --> 02:08:35,626 INDIVIDUAL RESEARCH PROJECT. 3206 02:08:35,626 --> 02:08:37,027 AND IF SUCCESSFUL WITH THIS 3207 02:08:37,027 --> 02:08:38,529 MODEL OF POOLING FUNDS TO 3208 02:08:38,529 --> 02:08:39,863 SUPPORT A RESEARCH PROJECT, IT 3209 02:08:39,863 --> 02:08:42,499 REALLY SETS THE STAGE FOR OTHER 3210 02:08:42,499 --> 02:08:43,634 POTENTIALLY LARGER INITIATIVES 3211 02:08:43,634 --> 02:08:47,571 DOWN THE ROAD THAT MAY MAKE MORE 3212 02:08:47,571 --> 02:08:49,339 SUBSTANTIAL IMPACT ON WOMEN'S 3213 02:08:49,339 --> 02:08:50,841 CARDIOVASCULAR HEALTH AND OTHER 3214 02:08:50,841 --> 02:08:54,678 CARDIOVASCULAR CONDITIONS. 3215 02:08:54,678 --> 02:08:55,879 SO WITH THAT, I'LL BE HEANS TO 3216 02:08:55,879 --> 02:08:59,716 ANSWER QUESTIONS OR GET YOUR 3217 02:08:59,716 --> 02:09:03,754 FEEDBACK. 3218 02:09:03,754 --> 02:09:05,756 >> AS I UNDERSTAND IT, THIS IS 3219 02:09:05,756 --> 02:09:08,592 $10 MILLION IN TOTAL FOR ONE 3220 02:09:08,592 --> 02:09:09,860 GRANT THAT CAN BE AWARDED? 3221 02:09:09,860 --> 02:09:10,160 >> YES. 3222 02:09:10,160 --> 02:09:16,166 >> AND IN RANDOM -- IN A RANDOM 3223 02:09:16,166 --> 02:09:16,400 COUNTRY? 3224 02:09:16,400 --> 02:09:18,502 >> YEAH, SO THE LEAD INSTITUTION 3225 02:09:18,502 --> 02:09:20,103 MUST BE IN ONE OF THE MEMBER 3226 02:09:20,103 --> 02:09:21,205 COUNTRIES, BUT IT'S EXPECTED TO 3227 02:09:21,205 --> 02:09:23,774 BE A PARTNERSHIP ACROSS 3228 02:09:23,774 --> 02:09:25,642 COUNTRIES AND AT LEAST WE ARE 3229 02:09:25,642 --> 02:09:27,077 HOPEFUL AND HAVE MADE IT CLEAR 3230 02:09:27,077 --> 02:09:29,480 WE THINK IT WOULD BE PREFERABLE 3231 02:09:29,480 --> 02:09:31,114 TO HAVE PARTNERSHIP WITH 3232 02:09:31,114 --> 02:09:31,982 INVESTIGATORS FROM LOW AND 3233 02:09:31,982 --> 02:09:37,788 MIDDLE INCOME COUNTRIES. 3234 02:09:37,788 --> 02:09:39,490 >> AND YOU SAID THE GROUP HAS 3235 02:09:39,490 --> 02:09:40,924 WORKED TOGETHER BUT THIS IS A 3236 02:09:40,924 --> 02:09:43,560 NEW MECHANISM FOR THE DIFFERENT 3237 02:09:43,560 --> 02:09:44,595 FOUNDATIONS TO WORK TOGETHER? 3238 02:09:44,595 --> 02:09:45,095 >> THAT'S RIGHT. 3239 02:09:45,095 --> 02:09:48,699 SO THE FIRST WORK STREAM IS A 3240 02:09:48,699 --> 02:09:50,534 MULTI-NATIONAL CLINICAL TRIALS 3241 02:09:50,534 --> 02:09:53,937 EFFORT, AND WHAT THAT HAS BEEN, 3242 02:09:53,937 --> 02:09:56,006 JUST TO BROAFL SUMMARIZE, IS 3243 02:09:56,006 --> 02:09:57,875 THERE IS A LETTER OF INTENT 3244 02:09:57,875 --> 02:09:59,042 PROCESS WHERE INVESTIGATORS WHO 3245 02:09:59,042 --> 02:10:01,912 WANT TO DO A MULTI-NATIONAL 3246 02:10:01,912 --> 02:10:03,313 CARDIOVASCULAR TRIAL SUBMIT A 3247 02:10:03,313 --> 02:10:05,816 LETTER OF INTENT, AND THAT GETS 3248 02:10:05,816 --> 02:10:06,617 REVIEWED BY A PANEL OF EXPERTS 3249 02:10:06,617 --> 02:10:08,252 WHO HAVE BEEN PUT TOGETHER 3250 02:10:08,252 --> 02:10:09,753 ACROSS THE MEMBER COUNTRIES, AND 3251 02:10:09,753 --> 02:10:13,156 THEN IT IS EVALUATED AND THE 3252 02:10:13,156 --> 02:10:15,759 GCRFF EITHER ENDORSES IT OR NOT. 3253 02:10:15,759 --> 02:10:18,161 AN ENDORSEMENT MEANS WE THINK 3254 02:10:18,161 --> 02:10:20,130 IT'S A GOOD IDEA, BUT YOU STILL 3255 02:10:20,130 --> 02:10:21,565 HAVE TO SUBMIT FOR FUNDING FROM 3256 02:10:21,565 --> 02:10:22,866 THE DIFFERENT MEMBER 3257 02:10:22,866 --> 02:10:24,101 ORGANIZATIONS AND MEMBER FUNDERS 3258 02:10:24,101 --> 02:10:25,469 FROM THE COUNTRIES YOU WANT TO 3259 02:10:25,469 --> 02:10:26,370 DO THE TRIAL IN. 3260 02:10:26,370 --> 02:10:27,804 AND EACH COUNTRY IS RESPONSIBLE 3261 02:10:27,804 --> 02:10:31,174 FOR THEIR OWN FUNDING, AND WE 3262 02:10:31,174 --> 02:10:32,476 HAVE NOW FUNDED A COUPLE OF 3263 02:10:32,476 --> 02:10:34,444 TRIALS THAT WAY IN PARTNERSHIP, 3264 02:10:34,444 --> 02:10:36,680 AND THAT'S BEEN REALLY EFFECTIVE 3265 02:10:36,680 --> 02:10:37,948 FOR US BECAUSE IT MEANS THE 3266 02:10:37,948 --> 02:10:39,483 TRIALS WE FUND ARE MORE LIKELY 3267 02:10:39,483 --> 02:10:42,553 TO BE HIGH IMPACT, REALLY AFFECT 3268 02:10:42,553 --> 02:10:44,988 GUIDELINES BECAUSE THEY'RE OF 3269 02:10:44,988 --> 02:10:46,557 MAJOR IMPORTANCE TO MORE THAN 3270 02:10:46,557 --> 02:10:48,759 ONE COUNTRY, AND WE'RE ALSO 3271 02:10:48,759 --> 02:10:50,294 SHARING THE FINANCIAL 3272 02:10:50,294 --> 02:10:51,595 RESPONSIBILITIES, SO WE GET A 3273 02:10:51,595 --> 02:10:53,764 LITTLE BIT BETTER BREAK IN TERMS 3274 02:10:53,764 --> 02:10:55,198 OF THE RETURN ON OUR INVESTMENT. 3275 02:10:55,198 --> 02:10:56,833 THAT'S BEEN EFFECTIVE, ALTHOUGH 3276 02:10:56,833 --> 02:11:03,407 THE DOWNSIDE HAS BEEN MULTIPLE 3277 02:11:03,407 --> 02:11:04,141 DIFFERENT REVIEW GROUPS, 3278 02:11:04,141 --> 02:11:05,475 DECISION PROCESSES, AND THAT HAS 3279 02:11:05,475 --> 02:11:06,443 SLOWED DOWN SOME OF THE PROGRESS 3280 02:11:06,443 --> 02:11:07,544 BECAUSE SOME ORGANIZATIONS ARE 3281 02:11:07,544 --> 02:11:08,645 MORE NIMBLE THAN OTHERS. 3282 02:11:08,645 --> 02:11:14,751 I WON'T NAME NAMES. 3283 02:11:14,751 --> 02:11:16,153 THE SECOND WORK STREAM, WE'VE 3284 02:11:16,153 --> 02:11:17,521 PULLED MONEY TOGETHER TO FUND A 3285 02:11:17,521 --> 02:11:20,324 SMALL KA DATA CHALLENGE PRIZE, 3286 02:11:20,324 --> 02:11:21,858 WHICH IS -- WE ARE SHARING OUR 3287 02:11:21,858 --> 02:11:24,061 DATA ON OUR RESEARCH FUNDING, 3288 02:11:24,061 --> 02:11:26,563 AND SO THIS WAY BY DOING THAT, 3289 02:11:26,563 --> 02:11:28,498 WE'RE ABLE TO GET AN IDEA 3290 02:11:28,498 --> 02:11:29,933 GLOBALLY WHAT ARE WE FUNDING, 3291 02:11:29,933 --> 02:11:32,002 BECAUSE WE KNOW WHAT WE'RE 3292 02:11:32,002 --> 02:11:34,304 FUNDING AND THEY KNOW WHAT 3293 02:11:34,304 --> 02:11:35,405 THEY'RE FUNDING BUT NOBODY HAS 3294 02:11:35,405 --> 02:11:36,840 REALLY TAKEN A LOOK AT THE TOTAL 3295 02:11:36,840 --> 02:11:38,575 PORTFOLIO OF THESE 13 DIFFERENT 3296 02:11:38,575 --> 02:11:39,109 FUNDERS. 3297 02:11:39,109 --> 02:11:41,178 SO WE'VE FUNDED A SMALL DATA 3298 02:11:41,178 --> 02:11:42,379 CHALLENGE PRIZE IN THAT SPACE. 3299 02:11:42,379 --> 02:11:44,147 THIS IS THE FIRST TIME WE'VE 3300 02:11:44,147 --> 02:11:46,216 TRIED TO DO SOMETHING RELATIVELY 3301 02:11:46,216 --> 02:11:48,418 BIG, AND EVEN $2 MILLION ISN'T 3302 02:11:48,418 --> 02:11:49,920 ALL THAT BIG, BUT RELATIVELY BIG 3303 02:11:49,920 --> 02:11:51,254 TO SEE IF WE CAN ACTUALLY WORK 3304 02:11:51,254 --> 02:11:54,191 THROUGH THE PROCESSES OF WORKING 3305 02:11:54,191 --> 02:11:56,360 TOGETHER AROUND A SINGLE REVIEW, 3306 02:11:56,360 --> 02:11:58,962 SHARED DECISION-MAKING, SHARED 3307 02:11:58,962 --> 02:12:01,365 FUNDING AND KABILITY. 3308 02:12:01,365 --> 02:12:03,767 ACCOUNTABILITY. 3309 02:12:03,767 --> 02:12:05,769 OTHER QUESTIONS, COMMENTS? 3310 02:12:05,769 --> 02:12:06,937 >> I THINK YOU ANSWERED. 3311 02:12:06,937 --> 02:12:08,238 I WAS JUST GOING TO ASK HOW THIS 3312 02:12:08,238 --> 02:12:14,378 IS REVIEWED, SO IS NIH JUST 3313 02:12:14,378 --> 02:12:16,880 FEEDING TO AN OUTSIDE REVIEW 3314 02:12:16,880 --> 02:12:18,448 COMMITTEE OR HOW DOES THAT WORK? 3315 02:12:18,448 --> 02:12:23,987 >> SO THERE IS A LEAD 3316 02:12:23,987 --> 02:12:25,489 ORGANIZATION, THE HEART AND 3317 02:12:25,489 --> 02:12:26,590 STROKE FOUNDATION OF CANADA HAS 3318 02:12:26,590 --> 02:12:27,357 AGREED TO BE THE LEAD 3319 02:12:27,357 --> 02:12:28,458 ORGANIZATION FOR THIS 3320 02:12:28,458 --> 02:12:31,061 INITIATIVE, AND SO THEY WILL BE 3321 02:12:31,061 --> 02:12:32,362 IMPANELING A REVIEW GROUP THAT 3322 02:12:32,362 --> 02:12:34,631 WOULD INCLUDE PEOPLE NOMINATED 3323 02:12:34,631 --> 02:12:36,967 BY THE MEMBER ORGANIZATION. 3324 02:12:36,967 --> 02:12:39,503 SO WE'LL BE NOMINATING REVIEWERS 3325 02:12:39,503 --> 02:12:43,073 TO BE ON THE PANEL THAT WILL BE 3326 02:12:43,073 --> 02:12:46,043 REVIEWING A PERSON WHO INTENDS 3327 02:12:46,043 --> 02:12:49,079 TO APPLY, THEY HAVE TO MANAGE 3328 02:12:49,079 --> 02:12:50,514 CONFLICTS AND ALL THAT, BUT TO 3329 02:12:50,514 --> 02:12:52,015 HAVE A SINGLE REVIEW GROUP 3330 02:12:52,015 --> 02:12:55,419 COMPRISECOMPRISED OF INTERNATIOL 3331 02:12:55,419 --> 02:12:56,787 EXPERTS IN CARDIOVASCULAR 3332 02:12:56,787 --> 02:12:57,187 WOMEN'S HEALTH. 3333 02:12:57,187 --> 02:12:58,488 AND THE HEART ASSOCIATION WILL 3334 02:12:58,488 --> 02:13:01,525 MANAGE THE MONEY. 3335 02:13:01,525 --> 02:13:02,526 BECAUSE THEY'RE ABLE TO TAKE 3336 02:13:02,526 --> 02:13:04,594 MONEY FROM MULTIPLE DIFFERENT 3337 02:13:04,594 --> 02:13:04,928 FOLKS. 3338 02:13:04,928 --> 02:13:07,130 WE WOULD HAVE DIFFICULTY AND 3339 02:13:07,130 --> 02:13:08,398 SOME OF THE OTHER FUNDERS WOULD 3340 02:13:08,398 --> 02:13:10,033 HAVE DIFFICULTY TAKING MONEY 3341 02:13:10,033 --> 02:13:14,971 FROM MULTIPLE DIFFERENT PEOPLE. 3342 02:13:14,971 --> 02:13:15,739 DIFFERENT ORGANIZATIONS. 3343 02:13:15,739 --> 02:13:17,908 IF NO OTHER COMMENTS -- HEART 3344 02:13:17,908 --> 02:13:19,209 ASSOCIATION IS REALLY GOOD AT 3345 02:13:19,209 --> 02:13:20,844 TAKING MONEY. 3346 02:13:20,844 --> 02:13:22,846 [LAUGHTER] 3347 02:13:22,846 --> 02:13:25,215 AND THEY DO GOOD THINGS WITH IT, 3348 02:13:25,215 --> 02:13:25,782 RIGHT, MERCEDES? 3349 02:13:25,782 --> 02:13:28,385 YEAH. 3350 02:13:28,385 --> 02:13:29,252 SO LET ME KNOW WHEN THE RATING 3351 02:13:29,252 --> 02:13:32,322 IS OVER AND WE'LL MOVE ON. 3352 02:13:32,322 --> 02:13:33,390 I THINK WE'RE PROBABLY DONE WITH 3353 02:13:33,390 --> 02:13:35,325 ALL THE QUESTIONS AND COMMENTS. 3354 02:13:35,325 --> 02:13:36,893 >> JUST A FEW PEOPLE ARE STILL 3355 02:13:36,893 --> 02:13:47,337 WORKING THEIR WAY THROUGH. 3356 02:13:52,375 --> 02:13:54,511 I THINK YOU CAN MOVE FORWARD, 3357 02:13:54,511 --> 02:13:54,778 DR. GOFF. 3358 02:13:54,778 --> 02:13:55,112 >> THANK YOU. 3359 02:13:55,112 --> 02:13:58,215 THE NEXT CONCEPT IS NUMBER 5038, 3360 02:13:58,215 --> 02:14:00,383 RENEWAL OF THE MAX WISE COMBINED 3361 02:14:00,383 --> 02:14:01,818 COHORT STUDY OF HIV IN THE 3362 02:14:01,818 --> 02:14:04,121 UNITED STATES. 3363 02:14:04,121 --> 02:14:05,622 THIS IS A RENEWAL RFA THAT WOULD 3364 02:14:05,622 --> 02:14:08,592 BE FUNDED THROUGH THE 3365 02:14:08,592 --> 02:14:11,194 U01 MECHANISM. 3366 02:14:11,194 --> 02:14:13,029 SO THIS INITIATIVE CALLS FOR 3367 02:14:13,029 --> 02:14:17,300 RENEWAL OF NHLBI SUPPORT FOR THE 3368 02:14:17,300 --> 02:14:19,803 MACS/WIHS COMBINED COHORT STUDY. 3369 02:14:19,803 --> 02:14:22,873 IN 2019 UNDER NHLBI LEADERSHIP, 3370 02:14:22,873 --> 02:14:24,307 NIH MERGED TWO LONGSTANDING 3371 02:14:24,307 --> 02:14:28,945 U.S.-BASED HIV COHORTS, THE 3372 02:14:28,945 --> 02:14:30,247 MULTICENTER AIDS COHORT STUDY OF 3373 02:14:30,247 --> 02:14:36,920 MEN AND THE WOMEN'S INTERAGENCY 3374 02:14:36,920 --> 02:14:39,990 HIV STUDY, I THINK IS WHAT THAT 3375 02:14:39,990 --> 02:14:43,927 WAS. 3376 02:14:43,927 --> 02:14:45,595 AND WE ORIENTED THEM ON A 3377 02:14:45,595 --> 02:14:46,997 PRIMARY FOCUS OF COMORBIDITIES 3378 02:14:46,997 --> 02:14:49,833 IN THE CONTEXT OF LONG-TERM 3379 02:14:49,833 --> 02:14:50,901 SURVIVAL WITH TREATED HIV. 3380 02:14:50,901 --> 02:14:52,702 BOTH OF THESE COHORT STUDIES 3381 02:14:52,702 --> 02:14:55,305 WERE STARTED IN THE ERA WHEN HIV 3382 02:14:55,305 --> 02:14:56,606 HAD MUCH FEWER EFFECTIVE 3383 02:14:56,606 --> 02:14:57,741 THERAPIES. 3384 02:14:57,741 --> 02:15:00,610 SO THIS LONGITUDINAL COHORT IS 3385 02:15:00,610 --> 02:15:02,412 CURRENTLY COMPRISED OF OVER 5300 3386 02:15:02,412 --> 02:15:04,714 LONG-TERM OR NEWLY ENROLLED 3387 02:15:04,714 --> 02:15:08,084 PARTICIPANTS AGED 35 AND OLDER, 3388 02:15:08,084 --> 02:15:09,186 HALF OF THEM ARE WOMEN AND THE 3389 02:15:09,186 --> 02:15:14,224 MAJORITY OF WHOM ARE GROUPS FROM 3390 02:15:14,224 --> 02:15:16,960 UNDERREPRESENTED IN MEDICAL 3391 02:15:16,960 --> 02:15:17,627 RESEARCH. 3392 02:15:17,627 --> 02:15:21,097 WE SEEK TO CONTINUE FOR AN 3393 02:15:21,097 --> 02:15:21,865 ADDITIONAL SEVEN YEARS AND 3394 02:15:21,865 --> 02:15:22,632 SEVERAL KEY AREAS OF SCIENCE 3395 02:15:22,632 --> 02:15:24,234 WILL BE EXPANDED IN RESPONSE TO 3396 02:15:24,234 --> 02:15:25,902 BOTH THE EVOLVING HIV KNOWLEDGE 3397 02:15:25,902 --> 02:15:27,637 BASE AND THE AGING OF THE 3398 02:15:27,637 --> 02:15:29,306 COHORT. 3399 02:15:29,306 --> 02:15:31,241 SO THE RENEWAL WILL EMPLOY A 3400 02:15:31,241 --> 02:15:34,644 LIMITED COMPETITION TO RETAIN A 3401 02:15:34,644 --> 02:15:35,946 GEOGRAPHICALLY DIVERSE GROUP OF 3402 02:15:35,946 --> 02:15:38,014 UP TO 13 CLINICAL RESEARCH SITE, 3403 02:15:38,014 --> 02:15:39,683 A LIMITED COMPETITION FOR A 3404 02:15:39,683 --> 02:15:41,418 SHARPLY REFOCUSED DATA ANALYSIS 3405 02:15:41,418 --> 02:15:43,820 AND DATA SHARING CENTER, AND AN 3406 02:15:43,820 --> 02:15:45,922 OPEN COMPETITION FOR A NEW 3407 02:15:45,922 --> 02:15:46,890 LEADERSHIP AND COORDINATING 3408 02:15:46,890 --> 02:15:50,126 CENTER. 3409 02:15:50,126 --> 02:15:52,529 THE NEW LEADERSHIP CENTER WILL 3410 02:15:52,529 --> 02:15:53,763 IMPROVE SCIENCE OF THE COHORT 3411 02:15:53,763 --> 02:15:57,133 AND CREATE AN ENHANCED STRUCTURE 3412 02:15:57,133 --> 02:16:00,403 FOR ANCILLARY STUDIES, WORK 3413 02:16:00,403 --> 02:16:01,371 GROUPS, OPERATIONAL -- AND 3414 02:16:01,371 --> 02:16:02,472 COMMUNITY IN PARTICIPANT 3415 02:16:02,472 --> 02:16:03,039 ENGAGEMENT. 3416 02:16:03,039 --> 02:16:04,541 WE ACTUALLY FOUND THAT WHEN WE 3417 02:16:04,541 --> 02:16:06,276 WERE MERGING THESE TWO COHORTS, 3418 02:16:06,276 --> 02:16:07,577 THERE WAS QUITE A BIT OF CULTURE 3419 02:16:07,577 --> 02:16:10,881 CHANGE AND A BUNCH OF CHANGE 3420 02:16:10,881 --> 02:16:14,684 MANAGEMENT, AND I THINK WE WISH 3421 02:16:14,684 --> 02:16:16,987 WE HAD STARTED A NEW LEADERSHIP 3422 02:16:16,987 --> 02:16:17,988 CENTER LAST ROUND, BUT HAVING 3423 02:16:17,988 --> 02:16:20,390 LEARNED THAT, WE WANT TO START 3424 02:16:20,390 --> 02:16:21,358 IT THIS ROUND. 3425 02:16:21,358 --> 02:16:24,661 THE MAX WISE HAS BEEN DESCRIBED 3426 02:16:24,661 --> 02:16:32,302 AS THE -- OF TH -- 40-YEAR 3427 02:16:32,302 --> 02:16:33,503 INVESTMENT IN ONE OF THE WORLD'S 3428 02:16:33,503 --> 02:16:35,372 MOST IMPORTANT SOURCES OF 3429 02:16:35,372 --> 02:16:36,473 RIGOROUS SCIENTIFIC KNOWLEDGE 3430 02:16:36,473 --> 02:16:39,943 ABOUT EVOLVING IMPACT OF HIV 3431 02:16:39,943 --> 02:16:41,478 INFECTION AND ITS COMORBIDITIES 3432 02:16:41,478 --> 02:16:46,716 ON HUMAN HEALTH. 3433 02:16:46,716 --> 02:16:47,817 SUSTAINING NHLBI'S LEADERSHIP OF 3434 02:16:47,817 --> 02:16:49,119 THE STUDY WILL CONTRIBUTE TO OUR 3435 02:16:49,119 --> 02:16:50,754 INFLUENCE ON THE GROWING FIELD 3436 02:16:50,754 --> 02:16:52,088 OF INTEGRATED SCIENCE, SEEKING 3437 02:16:52,088 --> 02:16:55,458 TO UNDERSTAND AND CONTROL HEART, 3438 02:16:55,458 --> 02:16:56,559 LUNG, BLOOD AND SLEEP CONDITIONS 3439 02:16:56,559 --> 02:16:59,062 OF VIRAL INFECTIONS. 3440 02:16:59,062 --> 02:17:01,331 AND WITH THAT, I'LL BE HAPPY TO 3441 02:17:01,331 --> 02:17:10,140 TAKE QUESTIONS AND COMMENTS. 3442 02:17:10,140 --> 02:17:12,409 >> I THINK IT'S REALLY IMPORTANT 3443 02:17:12,409 --> 02:17:14,477 THAT NHLBI CONTINUE INVOLVEMENT 3444 02:17:14,477 --> 02:17:17,113 IN THIS, PULMONARY AND 3445 02:17:17,113 --> 02:17:17,747 CARDIOVASCULAR COMPLICATIONS ARE 3446 02:17:17,747 --> 02:17:20,483 THE MAJOR COMORBIDITIES AND 3447 02:17:20,483 --> 02:17:21,785 SOURCES OF MORBIDITY AND 3448 02:17:21,785 --> 02:17:23,153 MORTALITY IN PEOPLE LIVING WITH 3449 02:17:23,153 --> 02:17:27,257 AIDS, WITH HIV, AND THE TOBACCO 3450 02:17:27,257 --> 02:17:30,627 EPIDEMIC, PEOPLE WITH HIV, IT'S 3451 02:17:30,627 --> 02:17:33,697 A MAJOR ISSUE. 3452 02:17:33,697 --> 02:17:35,365 I THINK IT'S A REALLY IMPORTANT 3453 02:17:35,365 --> 02:17:39,235 EA ONE THAT'S UNDERSTUDIED. 3454 02:17:39,235 --> 02:17:39,769 >> THANK YOU. 3455 02:17:39,769 --> 02:17:41,071 WE CERTAINLY AGREE, AND 3456 02:17:41,071 --> 02:17:44,007 APPRECIATE THE OPPORTUNITY AND 3457 02:17:44,007 --> 02:17:46,476 TRUST, REALLY, THAT NIH HAS 3458 02:17:46,476 --> 02:17:48,278 GIVEN IN US TO TAKE ON 3459 02:17:48,278 --> 02:17:49,379 STEWARDSHIP OF THIS RESOURCE. 3460 02:17:49,379 --> 02:17:50,680 WE THINK IT'S REALLY IMPORTANT. 3461 02:17:50,680 --> 02:17:52,449 WE HAVE A LOT OF IDEAS ABOUT HOW 3462 02:17:52,449 --> 02:17:53,416 TO LEVERAGE IT. 3463 02:17:53,416 --> 02:17:55,885 WE'RE BEGINNING TO DO 3464 02:17:55,885 --> 02:18:00,924 MULTI-OMICS IN MACS/WIHS, 3465 02:18:00,924 --> 02:18:02,559 CONTINUE TO DO MORE OF THAT AS 3466 02:18:02,559 --> 02:18:04,761 WE GO FORWARD, WE'LL HAVE SOME 3467 02:18:04,761 --> 02:18:07,731 COMPARATORS BETWEEN FOLKS IN 3468 02:18:07,731 --> 02:18:09,566 MACS/WIHS AND OUR OTHER COHORTS, 3469 02:18:09,566 --> 02:18:11,434 SO THERE'S JUST SO MANY 3470 02:18:11,434 --> 02:18:13,169 OPPORTUNITIES HERE TO SUPPORT 3471 02:18:13,169 --> 02:18:17,674 DISCOVERY SCIENCE. 3472 02:18:17,674 --> 02:18:18,541 PLEASE LET ME KNOW WHEN WE'RE 3473 02:18:18,541 --> 02:18:19,242 READY TO MOVE ON. 3474 02:18:19,242 --> 02:18:20,343 >> I THINK YOU CAN MOVE ON. 3475 02:18:20,343 --> 02:18:21,511 >> OKAY, THANK YOU. 3476 02:18:21,511 --> 02:18:25,849 THE NEXT CONCEPT IS 5042, 3477 02:18:25,849 --> 02:18:32,222 EMULATING ACCESS TO RESEARCH IN 3478 02:18:32,222 --> 02:18:38,428 RESIDENCY OR S STARR. 3479 02:18:38,428 --> 02:18:40,397 THIS RENEWAL OF WHICH NHLBI IS 3480 02:18:40,397 --> 02:18:42,832 THE LEAD INSTITUTE WITH 3481 02:18:42,832 --> 02:18:47,404 PARTICIPATION FROM NIAID, NCI, 3482 02:18:47,404 --> 02:18:51,574 NIA, AND NEI AND OTHER 3483 02:18:51,574 --> 02:18:52,108 PARTICIPATING INSTITUTES. 3484 02:18:52,108 --> 02:18:54,644 SO THE R38 IS AN INSTITUTIONAL 3485 02:18:54,644 --> 02:18:56,613 AWARD TO SUPPORT MENTORED 3486 02:18:56,613 --> 02:18:58,014 RESEARCH ACTIVITIES FOR 3487 02:18:58,014 --> 02:18:59,449 RESIDENTS WHO ARE EXPECTED TO 3488 02:18:59,449 --> 02:19:02,285 THEN PURSUE CAREERS AS PHYSICIAN 3489 02:19:02,285 --> 02:19:03,153 SCIENTISTS. 3490 02:19:03,153 --> 02:19:05,021 THE R38 SUPPORTS SPECIFIC 3491 02:19:05,021 --> 02:19:08,425 RESEARCH TOPICS WITHIN EACH 3492 02:19:08,425 --> 02:19:14,197 PARTICIPATING INSTIT INSTITUTE'S 3493 02:19:14,197 --> 02:19:17,600 INDIVIDUAL MISSIONS. 3494 02:19:17,600 --> 02:19:19,602 LESS THAN 5% OF ALL PHYSICIANS 3495 02:19:19,602 --> 02:19:24,174 HOLD AN MD PH.D., AND ALTHOUGH 3496 02:19:24,174 --> 02:19:26,142 MOST MD PH.D.s ARE ENGAGED IN 3497 02:19:26,142 --> 02:19:28,678 RESEARCH IN THEIR CAREERS, THE 3498 02:19:28,678 --> 02:19:32,882 MAJORITY OF SIT 10 SCIENTISTS IN 3499 02:19:32,882 --> 02:19:37,854 THE WORKFORCE ARE MADE UP OF 3500 02:19:37,854 --> 02:19:40,056 M.D.s WITHOUT A PH.D. 3501 02:19:40,056 --> 02:19:42,158 IT HIGHLIGHTED WHAT WE CALL AN 3502 02:19:42,158 --> 02:19:43,460 M.D. ONLY, ALTHOUGH IT'S NOT 3503 02:19:43,460 --> 02:19:47,430 JUST AN M.D., BUT AN M.D. ONLY 3504 02:19:47,430 --> 02:19:49,599 POOL ARE THE ONES THAT ARE 3505 02:19:49,599 --> 02:19:52,335 ENTERING ACADEMIC CAREERS AT A 3506 02:19:52,335 --> 02:19:52,769 DIMINISHING RATE. 3507 02:19:52,769 --> 02:19:54,304 YOU STILL SEE THAT PEOPLE WHO GO 3508 02:19:54,304 --> 02:19:57,040 TO THE POINT OF GETTING THE M.D. 3509 02:19:57,040 --> 02:20:00,210 PH.D., THEY TEND MOSTLY TO DO 3510 02:20:00,210 --> 02:20:01,744 RESEARCH, BUT THIS -- SHRINK IT. 3511 02:20:01,744 --> 02:20:03,046 THERE ARE A NUMBER OF 3512 02:20:03,046 --> 02:20:03,880 RECOMMENDATIONS THAT RESULTED 3513 02:20:03,880 --> 02:20:05,181 FROM THE WORKING GROUP THAT LED 3514 02:20:05,181 --> 02:20:09,385 TO THE CREATION OF THE R38 STARR 3515 02:20:09,385 --> 02:20:11,688 PROGRAM, INCLUDING THAT NIH'S 3516 02:20:11,688 --> 02:20:14,657 PILOT IDEAS TO TEST NOVEL 3517 02:20:14,657 --> 02:20:17,827 APPROACHES TO INPROVE SHORTING 3518 02:20:17,827 --> 02:20:19,762 RESEARCH TRAINING AND INTENSIFY 3519 02:20:19,762 --> 02:20:21,097 EFFORTS TO INCREASE DIVERSITY IN 3520 02:20:21,097 --> 02:20:22,732 THE WORKFORCE. 3521 02:20:22,732 --> 02:20:24,267 FOR RESIDENTS WHO ARE IN THE 3522 02:20:24,267 --> 02:20:26,002 STARR PROGRAM, THEY'RE SUPPORTED 3523 02:20:26,002 --> 02:20:27,770 AND MENTORED RESEARCH FOR 3524 02:20:27,770 --> 02:20:31,608 BETWEEN 12 AND 24 MONTHS AT 80% 3525 02:20:31,608 --> 02:20:32,475 EFFORT. 3526 02:20:32,475 --> 02:20:34,444 NHLBI HAS BEEN FUNDING R38 3527 02:20:34,444 --> 02:20:39,048 PROGRAMS SINCE ABOUT 2018. 3528 02:20:39,048 --> 02:20:40,783 AT PRESENT, WE SUPPORT 3529 02:20:40,783 --> 02:20:42,418 21 PROGRAMS FOR WHICH 158 3530 02:20:42,418 --> 02:20:45,188 RESIDENTS HAVE RECEIVED OR ARE 3531 02:20:45,188 --> 02:20:46,256 CURRENTLY RECEIVING RESEARCH 3532 02:20:46,256 --> 02:20:49,792 SUPPORT OF THESE 158 RESIDENTS, 3533 02:20:49,792 --> 02:20:52,729 47% ARE WOMEN, AND ABOUT 15% ARE 3534 02:20:52,729 --> 02:20:54,697 FROM UNDERREPRESENTED GROUPS. 3535 02:20:54,697 --> 02:20:59,269 BY SPECIALTY, THE MAJORITY ARE 3536 02:20:59,269 --> 02:21:01,604 IN INTERNAL MEDICINE, ABOUT 3537 02:21:01,604 --> 02:21:03,439 HALF, 42 IN PEDIATRICS, 23 3538 02:21:03,439 --> 02:21:04,641 SURGEONS, 12 FROM PATHOLOGY, AND 3539 02:21:04,641 --> 02:21:06,843 A SMALL NUMBER FROM FAMILY 3540 02:21:06,843 --> 02:21:08,711 MEDICINE AND EMERGENCY MEDICINE. 3541 02:21:08,711 --> 02:21:12,916 IN TERMS OF FUTURE SUCCESS, 42 3542 02:21:12,916 --> 02:21:14,484 OF THE RESIDENTS HAVE COMPLETED 3543 02:21:14,484 --> 02:21:16,219 THEIR R38 PROGRAM FOR AT LEAST 3544 02:21:16,219 --> 02:21:17,453 THREE YEARS. 3545 02:21:17,453 --> 02:21:19,522 AMONG THOSE 42, ABOUT HALF ARE 3546 02:21:19,522 --> 02:21:21,157 STILL IN CLINICAL TRAINING, 3547 02:21:21,157 --> 02:21:24,327 MOSTLY IN CLINICAL FELLOWSHIPS. 3548 02:21:24,327 --> 02:21:27,397 11 OR 26% HAVE RECEIVED AN NIH 3549 02:21:27,397 --> 02:21:32,368 AWARD SUCH AS A T32 OR K AWARD, 3550 02:21:32,368 --> 02:21:34,437 AND 9 OR 21% ARE CURRENTLY 3551 02:21:34,437 --> 02:21:36,873 ACADEMIC FACULTY AND PURSUING A 3552 02:21:36,873 --> 02:21:37,407 RESEARCH CAREER. 3553 02:21:37,407 --> 02:21:39,275 SO WE THINK THE EARLY RESULTS 3554 02:21:39,275 --> 02:21:42,212 ARE VERY PROMISING FOR THIS 3555 02:21:42,212 --> 02:21:42,879 PROGRAM. 3556 02:21:42,879 --> 02:21:45,181 WE ARE INTERESTED IN CONTINUING 3557 02:21:45,181 --> 02:21:49,118 TO SUPPORT TO SEE WHAT THE 3558 02:21:49,118 --> 02:21:50,320 LONGER TERM OUTCOMES WILL BE AND 3559 02:21:50,320 --> 02:21:51,421 LOOK FORWARD TO YOUR QUESTIONS 3560 02:21:51,421 --> 02:22:01,664 AND COMMENTS. 3561 02:22:02,332 --> 02:22:05,301 >> CAN I ASK A COUPLE QUESTIONS? 3562 02:22:05,301 --> 02:22:06,002 >> YES, PLEASE. 3563 02:22:06,002 --> 02:22:10,673 >> SO THE PROGRAM -- ADDITIONAL 3564 02:22:10,673 --> 02:22:14,277 18 MONTHS IS NOT -- [INAUDIBLE] 3565 02:22:14,277 --> 02:22:15,712 >> SO DIFFERENT PROGRAMS HAVE 3566 02:22:15,712 --> 02:22:16,746 DECIDED TO DO THAT DIFFERENTLY. 3567 02:22:16,746 --> 02:22:18,481 SOME ADD A YEAR, SOME INTEGRATE. 3568 02:22:18,481 --> 02:22:20,783 THE ONES THAT ADD A YEAR TEND TO 3569 02:22:20,783 --> 02:22:23,753 HAVE LONGER PERIODS OF RESEARCH. 3570 02:22:23,753 --> 02:22:25,688 THIS PROGRAM SUPPORTS 12 TO 24 3571 02:22:25,688 --> 02:22:27,190 MONTHS. 3572 02:22:27,190 --> 02:22:28,758 SO FOR THE PROGRAMS THAT ADD A 3573 02:22:28,758 --> 02:22:30,226 YEAR, THEY'RE TYPICALLY 3574 02:22:30,226 --> 02:22:32,395 INTEGRATING SOME RESEARCH TIME 3575 02:22:32,395 --> 02:22:34,464 AND ADDING RESEARCH TIME. 3576 02:22:34,464 --> 02:22:37,433 FOR THOSE THAT INTEGRATE, THEY 3577 02:22:37,433 --> 02:22:39,435 MOSTLY DON'T GET FAR BEYOND 12 3578 02:22:39,435 --> 02:22:40,837 MONTHS. 3579 02:22:40,837 --> 02:22:41,704 >> AND -- 3580 02:22:41,704 --> 02:22:43,873 >> THAT'S MY UNDERSTANDING, BUT 3581 02:22:43,873 --> 02:22:45,742 DAVID MAY BE OUT THERE, IF 3582 02:22:45,742 --> 02:22:48,244 YOU'RE ONLINE OR IN THE ROOM AND 3583 02:22:48,244 --> 02:22:49,545 I'M MISSTATING, PLEASE HOLLER 3584 02:22:49,545 --> 02:22:49,912 OUT. 3585 02:22:49,912 --> 02:22:50,747 >> I AM HERE. 3586 02:22:50,747 --> 02:22:52,282 THANK YOU, AND NO, YOU SAID THAT 3587 02:22:52,282 --> 02:22:55,251 EXACTLY RIGHT. 3588 02:22:55,251 --> 02:22:58,621 PROGRAMS -- ALL KIND OF 3589 02:22:58,621 --> 02:22:59,822 STRUCTURED SLIGHTLY DIFFERENTLY 3590 02:22:59,822 --> 02:23:01,124 AND AS YOU SAID, SOME ARE ABLE 3591 02:23:01,124 --> 02:23:03,126 TO DO ONE YEAR OF RESEARCH WB A 3592 02:23:03,126 --> 02:23:04,761 THREE-YEAR RESIDENCY. 3593 02:23:04,761 --> 02:23:06,162 IT COMPACTS THE REST OF THEIR 3594 02:23:06,162 --> 02:23:08,598 CLINICAL TIME INTO THEIR TWO 3595 02:23:08,598 --> 02:23:10,133 YEARS. 3596 02:23:10,133 --> 02:23:14,570 BUT SOME DO, ESPECIALLY IF THEY 3597 02:23:14,570 --> 02:23:16,339 DO 18 OR 24 MONTHS, WILL ADD ON 3598 02:23:16,339 --> 02:23:18,641 A FOURTH YEAR TO A THREE-YEAR 3599 02:23:18,641 --> 02:23:19,275 RESIDENCY, FOR EXAMPLE IF WE'RE 3600 02:23:19,275 --> 02:23:22,812 TALKING ABOUT MEDICINE AND 3601 02:23:22,812 --> 02:23:23,680 PEDIATRICS, WHICH MAKE UP THE 3602 02:23:23,680 --> 02:23:24,380 BULK OF THE PROGRAMS. 3603 02:23:24,380 --> 02:23:27,717 >> ONE MORE QUESTION. 3604 02:23:27,717 --> 02:23:31,054 JUST IN TERMS OF DIVERSITY, ARE 3605 02:23:31,054 --> 02:23:32,255 INTERNATIONAL GRADUATES ELIGIBLE 3606 02:23:32,255 --> 02:23:34,624 OR DOES THIS HAVE A U.S. 3607 02:23:34,624 --> 02:23:35,491 CITIZENSHIP REQUIREMENT? 3608 02:23:35,491 --> 02:23:36,726 >> DAVID, DO YOU KNOW THE ANSWER 3609 02:23:36,726 --> 02:23:37,060 TO THAT? 3610 02:23:37,060 --> 02:23:38,227 I DON'T KNOW THE ANSWER TO THAT. 3611 02:23:38,227 --> 02:23:39,662 >> I DON'T BELIEVE THERE IS -- 3612 02:23:39,662 --> 02:23:42,098 THAT WE PUT A U.S. CITIZENSHIP 3613 02:23:42,098 --> 02:23:43,066 REQUIREMENT ON IT. 3614 02:23:43,066 --> 02:23:45,034 NO. 3615 02:23:45,034 --> 02:23:48,104 NO, JUST THAT THEY'RE IN 3616 02:23:48,104 --> 02:23:49,772 RESIDENCY, BUT WE DON'T 3617 02:23:49,772 --> 02:23:51,941 STIPULATE RESIDENCY STATUS FOR 3618 02:23:51,941 --> 02:23:53,242 THIS, I'M PRETTY SURE. 3619 02:23:53,242 --> 02:23:56,813 I CAN ALWAYS CONFIRM, BUT NO, IT 3620 02:23:56,813 --> 02:23:58,314 WAS NOT OUR INTENTION TO DO 3621 02:23:58,314 --> 02:23:59,749 THAT, AT LEAST. 3622 02:23:59,749 --> 02:24:00,616 >> THAT'S WONDERFUL. 3623 02:24:00,616 --> 02:24:04,654 THANK YOU SO MUCH. 3624 02:24:04,654 --> 02:24:05,688 >> ANY OTHER QUESTIONS OR 3625 02:24:05,688 --> 02:24:07,090 COMMENTS OR WE CAN MOVE ON TO 3626 02:24:07,090 --> 02:24:17,233 RATINGS? 3627 02:24:31,447 --> 02:24:32,782 >> MAYBE JUST ANOTHER 3628 02:24:32,782 --> 02:24:33,616 30 SECONDS, WE'LL BE ABLE TO 3629 02:24:33,616 --> 02:24:43,760 MOVE ON. 3630 02:24:48,164 --> 02:24:48,664 OKAY, DR. GOFF. 3631 02:24:48,664 --> 02:24:49,332 YOU CAN MOVE ON. 3632 02:24:49,332 --> 02:24:51,167 >> THANK YOU. 3633 02:24:51,167 --> 02:24:53,169 THE FINAL CONCEPT WE HAVE FROM 3634 02:24:53,169 --> 02:24:57,640 DCBS IS NUMBER 5044 TITLED WHOLE 3635 02:24:57,640 --> 02:24:59,509 PERSON RESEARCH AND COORDINATION 3636 02:24:59,509 --> 02:25:00,510 CENTER. 3637 02:25:00,510 --> 02:25:04,013 THIS IS SECONDARY PARTICIPATION 3638 02:25:04,013 --> 02:25:08,251 AND THEN TRANS-NIH RFA TO FUND A 3639 02:25:08,251 --> 02:25:12,088 U24 MECHANISM LED BY NCCIH. 3640 02:25:12,088 --> 02:25:13,856 SO THE GOAL OF THIS INITIATIVE 3641 02:25:13,856 --> 02:25:16,459 IS TO BUILD AN INTEGRATED 3642 02:25:16,459 --> 02:25:17,894 FRAMEWORK WITH MOLECULAR STUDIES 3643 02:25:17,894 --> 02:25:20,296 AT ONE END OF THE SPECTRUM TO 3644 02:25:20,296 --> 02:25:22,398 PATIENT AND SOCIETAL LEVEL DATA, 3645 02:25:22,398 --> 02:25:23,766 INCLUDING SOCIAL DETERMINANTS OF 3646 02:25:23,766 --> 02:25:24,801 HEALTH AND CULTURAL FACTORS AT 3647 02:25:24,801 --> 02:25:27,103 THE OTHER END OF THE SPECTRUM. 3648 02:25:27,103 --> 02:25:28,838 STARTING IN THE MIDDLE 3649 02:25:28,838 --> 02:25:32,275 PHYSIOLOGICAL LAYER OR ORGAN AND 3650 02:25:32,275 --> 02:25:34,677 SYSTEM-LEVEL FUNCTIONING. 3651 02:25:34,677 --> 02:25:35,945 THE NOTICE OF FUNDING 3652 02:25:35,945 --> 02:25:37,947 OPPORTUNITY WILL INCLUDE FOUR 3653 02:25:37,947 --> 02:25:40,149 BASIC SCIENCE COMPONENTS OR FOUR 3654 02:25:40,149 --> 02:25:42,218 BASIC CORE COMPONENTS, I SHOULD 3655 02:25:42,218 --> 02:25:43,553 SAY, SCIENTIFIC LEADERSHIP, 3656 02:25:43,553 --> 02:25:45,755 DOMAIN MAPPING AND COMMON DATA 3657 02:25:45,755 --> 02:25:47,490 ELEMENTS, DATA SCIENCE AND 3658 02:25:47,490 --> 02:25:49,459 MODELING, AND A COORDINATION 3659 02:25:49,459 --> 02:25:49,759 CORE. 3660 02:25:49,759 --> 02:25:52,261 SO FAR, 15 INSTITUTES, CENTERS 3661 02:25:52,261 --> 02:25:54,163 AND OFFICES ACROSS NIH HAVE 3662 02:25:54,163 --> 02:25:56,265 AGREED TO PARTICIPATE IN THIS 3663 02:25:56,265 --> 02:25:58,434 NEW EFFORT. 3664 02:25:58,434 --> 02:25:59,969 THE U24 MECHANISM IS PLANNED TO 3665 02:25:59,969 --> 02:26:03,072 BE DEPLOYED IN TWO PHASES. 3666 02:26:03,072 --> 02:26:04,607 ONE COORDINATING CENTER WILL BE 3667 02:26:04,607 --> 02:26:05,241 FUNDED. 3668 02:26:05,241 --> 02:26:06,776 THE GOAL OF THE FIRST PHASE, 3669 02:26:06,776 --> 02:26:10,413 YEARS ONE AND TWO OF THE AWARD, 3670 02:26:10,413 --> 02:26:13,182 IS TO BUILD A HUMAN WHOLE PERSON 3671 02:26:13,182 --> 02:26:14,150 CONCEPT MAP OF WHAT WE ALREADY 3672 02:26:14,150 --> 02:26:15,785 KNOW ABOUT MAJOR PHYSIOLOGICAL 3673 02:26:15,785 --> 02:26:16,052 FUNCTIONS. 3674 02:26:16,052 --> 02:26:17,987 IT WILL BE BUILT BY A TEAM OF 3675 02:26:17,987 --> 02:26:19,655 EXPERTS REPRESENTING VARIOUS 3676 02:26:19,655 --> 02:26:21,057 PHYSIOLOGICAL SYSTEMS. 3677 02:26:21,057 --> 02:26:23,159 THE NEXT STEP IN PHASE ONE WILL 3678 02:26:23,159 --> 02:26:24,794 BE TO IDENTIFY COMMON DATA 3679 02:26:24,794 --> 02:26:26,762 ELEMENTS FOR EACH PHYSIOLOGICAL 3680 02:26:26,762 --> 02:26:27,763 FUNCTION. 3681 02:26:27,763 --> 02:26:29,732 IN PHASE TWO, WHICH IS 3682 02:26:29,732 --> 02:26:30,933 ENVISIONED AS YEARS THREE 3683 02:26:30,933 --> 02:26:32,902 THROUGH FIVE, THE CONCEPT MAP 3684 02:26:32,902 --> 02:26:34,971 WILL BE POPULATED WITH DATA FROM 3685 02:26:34,971 --> 02:26:36,539 EXISTING INITIATIVES AND COHORT 3686 02:26:36,539 --> 02:26:38,374 STUDIES SUCH AS ALL-OF-US AND 3687 02:26:38,374 --> 02:26:39,909 OUR TOP MED PROGRAM. 3688 02:26:39,909 --> 02:26:41,677 THIS DATA POPULATED NETWORK 3689 02:26:41,677 --> 02:26:44,981 WOULD THEN BE USED TO BUILD AND 3690 02:26:44,981 --> 02:26:47,483 TEST INSILICO OR COMPUTER 3691 02:26:47,483 --> 02:26:50,019 GENERATED MODELS, FOCUSING ON 3692 02:26:50,019 --> 02:26:51,320 LIFESTYLE INTERVENTIONS TO 3693 02:26:51,320 --> 02:26:53,723 SUPPORT WHOLE PERSON HEALTH. 3694 02:26:53,723 --> 02:26:55,958 IN FUTURE PROJECTS, THIS CAN BE 3695 02:26:55,958 --> 02:26:59,262 USED TO MODEL DISEASE 3696 02:26:59,262 --> 02:27:00,029 PATHOPHYSIOLOGY AND THE EFFECTS 3697 02:27:00,029 --> 02:27:01,531 OF TREATMENTS. 3698 02:27:01,531 --> 02:27:03,499 THIS NOFO WOULD PROVIDE 3699 02:27:03,499 --> 02:27:04,467 OPPORTUNITY TO INCREASE OUR 3700 02:27:04,467 --> 02:27:06,335 RETURN ON INVESTMENT AND OUR 3701 02:27:06,335 --> 02:27:07,970 BIODATA CATALYST PROGRAM, TOP 3702 02:27:07,970 --> 02:27:09,639 MED, LUNG MAP, THE NATIONAL 3703 02:27:09,639 --> 02:27:12,475 SLEEP RESEARCH RESOURCES 3704 02:27:12,475 --> 02:27:16,312 PROGRAM, THE -- AND OUR SEAL 3705 02:27:16,312 --> 02:27:18,948 PROGRAM AMONG OUR OTHER NHLBI 3706 02:27:18,948 --> 02:27:19,282 INVESTMENTS. 3707 02:27:19,282 --> 02:27:20,249 THERE WILL ALSO BE OPPORTUNITIES 3708 02:27:20,249 --> 02:27:22,018 FOR US TO BRING NHLBI RESEARCH 3709 02:27:22,018 --> 02:27:25,955 PRIORITIES INTO THIS NIH-WIDE 3710 02:27:25,955 --> 02:27:27,056 INITIATIVE AND OUR INVESTIGATORS 3711 02:27:27,056 --> 02:27:28,724 WILL BE ABLE TO UTILIZE THE 3712 02:27:28,724 --> 02:27:30,026 RESOURCES AND BE ABLE TO JOIN 3713 02:27:30,026 --> 02:27:31,227 THIS WHOLE PERSON RESEARCH 3714 02:27:31,227 --> 02:27:34,297 COMMUNITY. 3715 02:27:34,297 --> 02:27:35,731 NCCIH WILL PROVIDE MORE THAN 3716 02:27:35,731 --> 02:27:41,304 HALF OF THE OVERALL FUNDING, 3717 02:27:41,304 --> 02:27:43,172 PROPOSED CONTRIBUTION OF 100,000 3718 02:27:43,172 --> 02:27:44,373 PER YEAR FOR FIVE YEARS OR A 3719 02:27:44,373 --> 02:27:46,342 TOTAL OF $500,000 WOULD 3720 02:27:46,342 --> 02:27:48,210 CONSTITUTE ABOUT 4% OF THE 3721 02:27:48,210 --> 02:27:50,079 PROJECT'S OVERALL BUDGET. 3722 02:27:50,079 --> 02:27:52,181 IF SUCCESSFUL WITH THIS REALLY 3723 02:27:52,181 --> 02:27:57,019 MODEST INVESTMENT, YO WE THINK Y 3724 02:27:57,019 --> 02:27:57,887 SCIENTIFIC DISCOVERIES ARE 3725 02:27:57,887 --> 02:27:59,755 POSSIBLE WITH COLLABORATION FROM 3726 02:27:59,755 --> 02:28:00,856 NCCIH AND OTHER INSTITUTES. 3727 02:28:00,856 --> 02:28:02,558 AT THIS POINT, I'D BE HAPPY TO 3728 02:28:02,558 --> 02:28:03,159 TAKE YOUR QUESTIONS AND 3729 02:28:03,159 --> 02:28:13,336 COMMENTS. 3730 02:28:14,270 --> 02:28:19,475 OR WE CAN MOVE TO RATINGS. 3731 02:28:19,475 --> 02:28:26,082 AND I'LL PASS THE BATON BACK TO 3732 02:28:26,082 --> 02:28:28,517 DR. PRENGER. 3733 02:28:28,517 --> 02:28:29,352 >> THANK YOU VERY MUCH. 3734 02:28:29,352 --> 02:28:36,125 WHILE YOU FINISH, WE'LL MOVE ON 3735 02:28:36,125 --> 02:28:46,669 TO -- DR. MENSAH CAN BEGIN THAT. 3736 02:28:50,106 --> 02:28:53,109 >> OKAY, DR. MENSAH, IT'S ALL 3737 02:28:53,109 --> 02:28:54,210 YOURS. 3738 02:28:54,210 --> 02:28:58,481 >> THANK YOU VERY MUCH. 3739 02:28:58,481 --> 02:28:59,448 WE'RE ALMOST AT THE END HERE. 3740 02:28:59,448 --> 02:29:01,550 IT'S MY PLEASURE TO REPRESENT 3741 02:29:01,550 --> 02:29:03,052 THE DIVISION OF EXTRAMURAL 3742 02:29:03,052 --> 02:29:04,954 RESEARCH ACTIVITIES IN 3743 02:29:04,954 --> 02:29:07,056 PRESENTING THE LAST SEVEN 3744 02:29:07,056 --> 02:29:10,326 CONCEPTS. 3745 02:29:10,326 --> 02:29:12,294 THE FIRST TWO CONCEPT MEMBERS 3746 02:29:12,294 --> 02:29:20,302 5028 5028 AND 5029 ARE RENEWAL 3747 02:29:20,302 --> 02:29:21,604 RFAs. 3748 02:29:21,604 --> 02:29:23,906 THEY'RE INTENDED TO ACCELERATE 3749 02:29:23,906 --> 02:29:24,340 COMMERCIALIZATION OF 3750 02:29:24,340 --> 02:29:25,474 TECHNOLOGIES FOR HEART, LUNG, 3751 02:29:25,474 --> 02:29:27,443 BLOOD AND SLEEP DISORDERS AND 3752 02:29:27,443 --> 02:29:28,010 DISEASES. 3753 02:29:28,010 --> 02:29:30,913 THEY USE THE R24 MECHANISM. 3754 02:29:30,913 --> 02:29:32,281 I'D LIKE TO BEGIN BY GIVING 3755 02:29:32,281 --> 02:29:35,718 CREDIT TO DR. JANE -- FROM THE 3756 02:29:35,718 --> 02:29:37,553 DIVISION OF EXTRAMURAL RESEARCH 3757 02:29:37,553 --> 02:29:39,622 ACTIVITY, WHO HAS SHEPHERDED 3758 02:29:39,622 --> 02:29:44,427 THESE PROGRAMS THROUGH OUR -- 3759 02:29:44,427 --> 02:29:47,930 THAN NORMAL PROCESSES. 3760 02:29:47,930 --> 02:29:51,534 CONCEPT 5028 SUPPORTS THE BRIDGE 3761 02:29:51,534 --> 02:29:54,270 AWARDS WHIEK 5029 PROVIDES SMALL 3762 02:29:54,270 --> 02:29:54,704 MARKET AWARDS. 3763 02:29:54,704 --> 02:29:55,705 I'LL PRESENT BOTH OF THEM 3764 02:29:55,705 --> 02:29:56,806 TOGETHER BUT THEN GIVE YOU TIME 3765 02:29:56,806 --> 02:30:00,976 TO RATE THEM INDEPENDENTLY. 3766 02:30:00,976 --> 02:30:02,611 THE PHASE 2B PROGRAM AS YOU KNOW 3767 02:30:02,611 --> 02:30:09,752 IS DESIGNED TO -- TECHNOLOGY 3768 02:30:09,752 --> 02:30:12,788 FUNDING BETWEEN THE EAND OF AN 3769 02:30:12,788 --> 02:30:15,257 SBIR AWARD AND ACQUISITION OF 3770 02:30:15,257 --> 02:30:16,625 THIRD PARTY -- NEEDED TO 3771 02:30:16,625 --> 02:30:19,061 ACCOMPLISH REGULATORY MILESTONES 3772 02:30:19,061 --> 02:30:22,932 AND ADDITIONAL DE-RISKING AS 3773 02:30:22,932 --> 02:30:23,966 SMALL BUSINESS ORGANIZATIONS 3774 02:30:23,966 --> 02:30:25,501 COMMERCIALIZE THEIR PRODUCTS. 3775 02:30:25,501 --> 02:30:27,369 THE TWO KEY GOALS OF THE PROGRAM 3776 02:30:27,369 --> 02:30:32,641 ARE TO PROVIDE FUNDING FOR 3777 02:30:32,641 --> 02:30:34,710 CAPITAL-INTENSIVE STAGE PRODUCT 3778 02:30:34,710 --> 02:30:36,579 DEVELOPMENT ACTIVITIES THAT ARE 3779 02:30:36,579 --> 02:30:38,981 REQUIRED TO ADVANCE PROJECTS TO 3780 02:30:38,981 --> 02:30:40,549 REGULATORY APPROVAL AND 3781 02:30:40,549 --> 02:30:43,285 COMMERCIALIZATION. 3782 02:30:43,285 --> 02:30:45,154 THE SECOND IS TO ENCOURAGE 3783 02:30:45,154 --> 02:30:47,923 PUBLIC PRIVATE PARTNERSHIPS THAT 3784 02:30:47,923 --> 02:30:50,092 ENHANCE THE RETURN ON 3785 02:30:50,092 --> 02:30:51,494 INVESTMENTS WE MAKE IN 3786 02:30:51,494 --> 02:30:55,765 PREVIOUSLY FUNDED TECHNOLOGIES. 3787 02:30:55,765 --> 02:30:58,501 SINCE ITS RELEASE BACK IN 2013, 3788 02:30:58,501 --> 02:31:01,070 THE PROGRAM HAS FUNDED 44 AWARDS 3789 02:31:01,070 --> 02:31:05,841 OUT OF A TOTAL OF 117 3790 02:31:05,841 --> 02:31:06,509 APPLICATIONS SUBMITTED. 3791 02:31:06,509 --> 02:31:07,877 THIS FUNDING IS PARTICULARLY 3792 02:31:07,877 --> 02:31:11,580 CRUCIAL IN TODAY'S INVESTMENT 3793 02:31:11,580 --> 02:31:13,215 LANDSCAPE WHERE EXTERNAL FUNDERS 3794 02:31:13,215 --> 02:31:16,418 HAVE BECOME INCREASINGLY 3795 02:31:16,418 --> 02:31:17,153 RISK-AVERSE. 3796 02:31:17,153 --> 02:31:18,487 PROGRAM EVALUATION SHOWS THAT 3797 02:31:18,487 --> 02:31:21,023 THE FUNDED PHASE 2B BRIDGE 3798 02:31:21,023 --> 02:31:22,992 AWARDEES HAVE ACTUALLY BEEN MORE 3799 02:31:22,992 --> 02:31:24,960 SUCCESSFUL AT ACHIEVING 3800 02:31:24,960 --> 02:31:26,729 MILESTONES AND ATTRACTING 3801 02:31:26,729 --> 02:31:29,031 INVESTMENT COMPARED TO THEIR 3802 02:31:29,031 --> 02:31:29,899 UNFUNDED COUNTERPARTS. 3803 02:31:29,899 --> 02:31:32,101 FOR EXAMPLE, THE EVALUATION 3804 02:31:32,101 --> 02:31:35,271 SHOWS THAT ABOUT A THIRD OF THE 3805 02:31:35,271 --> 02:31:37,807 PHASE 2B FUNDED PROJECTS HAVE 3806 02:31:37,807 --> 02:31:40,109 REACHED THE COMMERCIAL STAGE. 3807 02:31:40,109 --> 02:31:41,443 ANOTHER 20% HAVE SUCCESSFULLY 3808 02:31:41,443 --> 02:31:46,782 BEEN ACQUIRED BY OTHER 3809 02:31:46,782 --> 02:31:47,049 COMPANIES. 3810 02:31:47,049 --> 02:31:48,217 OUT OF 31 TECHNOLOGIES THAT ARE 3811 02:31:48,217 --> 02:31:49,985 IN DEVELOPMENT, ABOUT 40% OF 3812 02:31:49,985 --> 02:31:51,453 THEM ARE IN CLINICAL SETTINGS. 3813 02:31:51,453 --> 02:31:54,623 SO BASED ON THE RESULTS OF THESE 3814 02:31:54,623 --> 02:31:57,393 EVALUATIONS, WE REMAIN VERY 3815 02:31:57,393 --> 02:31:58,260 ENTHUSIASTIC ABOUT THE PROGRAM, 3816 02:31:58,260 --> 02:32:00,262 AND LOOK FORWARD TO COUNCIL'S 3817 02:32:00,262 --> 02:32:02,298 SUPPORT FOR THE RENEWAL. 3818 02:32:02,298 --> 02:32:09,238 LET ME TELL YOU ABOUT A SECOND, 3819 02:32:09,238 --> 02:32:09,438 5029. 3820 02:32:09,438 --> 02:32:10,840 NOW WHILE THE BRIDGE AWARD YOU 3821 02:32:10,840 --> 02:32:12,208 JUST HEARD ABOUT SUPPORTS SMALL 3822 02:32:12,208 --> 02:32:14,243 BUSINESSES WORKING IN ALL OF 3823 02:32:14,243 --> 02:32:18,514 NHLBI'S MISSION AREAS, A SMALL 3824 02:32:18,514 --> 02:32:19,615 MARKET RFA IS SPECIFICALLY 3825 02:32:19,615 --> 02:32:21,483 DESIGNED TO SUPPORT COMPANIES 3826 02:32:21,483 --> 02:32:24,653 DEVELOPING TECHNOLOGIES WITH 3827 02:32:24,653 --> 02:32:25,521 SMALL COMMERCIAL MARKETS OR 3828 02:32:25,521 --> 02:32:27,289 THOSE DEVELOPING TECHNOLOGIES 3829 02:32:27,289 --> 02:32:30,359 FOR PEDIATRIC IMPLICATIONS. 3830 02:32:30,359 --> 02:32:32,228 COMPANIES FOCUSED ON 3831 02:32:32,228 --> 02:32:34,964 TECHNOLOGIES WITH SMALL 3832 02:32:34,964 --> 02:32:36,732 COMMERCIAL MARKETS FILL A VERY 3833 02:32:36,732 --> 02:32:37,600 IMPORTANT CLINICAL NEED AND, 3834 02:32:37,600 --> 02:32:40,669 VERY OFTEN, THEY ARE THE FIRST 3835 02:32:40,669 --> 02:32:42,204 AND HARDEST HIT WHEN INVESTORS 3836 02:32:42,204 --> 02:32:44,173 DOUBLE DOWN ON BIG WINNERS IN 3837 02:32:44,173 --> 02:32:47,376 THE PORTFOLIO. 3838 02:32:47,376 --> 02:32:51,313 ADDITIONALLY, COMPANIES FOCUSING 3839 02:32:51,313 --> 02:32:52,982 ON -- DRUG DEVELOPMENT AND 3840 02:32:52,982 --> 02:32:55,184 PEDIATRIC DEVICE MANUFACTURERS 3841 02:32:55,184 --> 02:32:57,253 OFTEN NEGATIVELY AFFECTED BY THE 3842 02:32:57,253 --> 02:33:00,890 TENDENCY TO AVOID RISKY 3843 02:33:00,890 --> 02:33:02,524 INVESTMENTS. 3844 02:33:02,524 --> 02:33:07,897 HOWEVER, -- VALUABLE CLINICAL 3845 02:33:07,897 --> 02:33:09,531 NEED, DESIGNED FOR I A DULT 3846 02:33:09,531 --> 02:33:11,300 PATIENTS AND VERY ILL SUITED FOR 3847 02:33:11,300 --> 02:33:14,036 NEONATAL AND PEDIATRIC PATIENTS. 3848 02:33:14,036 --> 02:33:15,471 ADDITIONALLY, SMALL MARKET 3849 02:33:15,471 --> 02:33:19,975 AWARDEES ARE REQUIRED TO 3850 02:33:19,975 --> 02:33:20,910 SECURE -- THEY'RE REQUIRED TO 3851 02:33:20,910 --> 02:33:22,311 MAKE A ONE TO THREE MATCH RATHER 3852 02:33:22,311 --> 02:33:25,147 THAN A ONE TO ONE MATCH, SO THE 3853 02:33:25,147 --> 02:33:25,814 BRIDGE AWARDS. 3854 02:33:25,814 --> 02:33:29,184 FOR INSTANCE, IF A COMPANY 3855 02:33:29,184 --> 02:33:30,052 RECEIVES $3 MILLION AWARD FROM 3856 02:33:30,052 --> 02:33:31,720 NHLBI, THEY MUST BRING IN AT 3857 02:33:31,720 --> 02:33:33,889 LEAST A MILLION FROM OTHER 3858 02:33:33,889 --> 02:33:37,960 PRIVATE INVESTORS OR SOURCES 3859 02:33:37,960 --> 02:33:40,896 OF -- FUNDING. 3860 02:33:40,896 --> 02:33:43,666 SINCE ITS INCEPTION IN 2014, THE 3861 02:33:43,666 --> 02:33:45,100 PROGRAM HAS YIELDED 14 AWARDS 3862 02:33:45,100 --> 02:33:50,572 FROM A TOTAL OF 49 APPLICATIONS. 3863 02:33:50,572 --> 02:33:56,178 2023 EVALUATION SHOWS THAT -- AT 3864 02:33:56,178 --> 02:33:58,380 ACHIEVING CONVENTIONAL 3865 02:33:58,380 --> 02:34:00,249 MILESTONES AND -- INVESTMENTS 3866 02:34:00,249 --> 02:34:01,850 COMPARED TO THE UNFUNDED 3867 02:34:01,850 --> 02:34:05,487 COUNTERPARTS. 3868 02:34:05,487 --> 02:34:06,588 IMPORTANTLY, THE ANALYSIS SHOWS 3869 02:34:06,588 --> 02:34:08,457 THAT FOR EVERY DOLLAR OF PHASE 3870 02:34:08,457 --> 02:34:11,060 2B FUNDS THAT NHLBI INVESTED IN 3871 02:34:11,060 --> 02:34:13,629 THE PROGRAM, THE AWARDED 3872 02:34:13,629 --> 02:34:15,464 COMPANIES RAISED 11 MILLION -- 3873 02:34:15,464 --> 02:34:18,701 I'M SORRY -- THAT WOULD HAVE 3874 02:34:18,701 --> 02:34:21,770 BEEN GREAT RETURN ON INVESTMENT. 3875 02:34:21,770 --> 02:34:23,739 $11 FOR EVERY DOLLAR WE INVESTED 3876 02:34:23,739 --> 02:34:27,376 IN TERMS OF FULL-ON FUNDING 3877 02:34:27,376 --> 02:34:29,011 DURING AND AFTER AWARD TO 3878 02:34:29,011 --> 02:34:32,614 DEVELOP THEIR TECHNOLOGIES. 3879 02:34:32,614 --> 02:34:34,616 BASED ON THESE RESULTS AND THESE 3880 02:34:34,616 --> 02:34:35,718 EVALUATIONS, WE'RE VERY 3881 02:34:35,718 --> 02:34:36,585 ENTHUSIASTIC OF THIS M PRA, AND 3882 02:34:36,585 --> 02:34:41,890 WEPROGRAM ANDWE LOOK FORWARD TOS 3883 02:34:41,890 --> 02:34:44,626 SUPPORT OF THE RENEWAL OF THE 3884 02:34:44,626 --> 02:34:46,628 SMALL MARKET RFA TO HELP 3885 02:34:46,628 --> 02:34:47,262 COMPANIES BRING THEIR PRODUCTS 3886 02:34:47,262 --> 02:34:48,263 TO PEDIATRIC PATIENTS AND TO 3887 02:34:48,263 --> 02:34:50,432 THOSE WITH RARE DISEASES. 3888 02:34:50,432 --> 02:34:52,101 I'LL PAUSE HERE FOR YOUR 3889 02:34:52,101 --> 02:34:55,070 QUESTIONS, YOUR COMMENTS, AND 3890 02:34:55,070 --> 02:34:57,239 THEN RATE THE TWO PROGRAMS. 3891 02:34:57,239 --> 02:35:01,176 AGAIN, THE NUMBERS ARE 5028 AND 3892 02:35:01,176 --> 02:35:07,549 5029. 3893 02:35:07,549 --> 02:35:07,783 PLEASE. 3894 02:35:07,783 --> 02:35:10,185 >> YOU TALK ABOUT HOW SUCCESSFUL 3895 02:35:10,185 --> 02:35:11,387 THESE APPLICANTS ARE IN GETTING 3896 02:35:11,387 --> 02:35:13,288 FUNDING, AND I'M JUST CURIOUS 3897 02:35:13,288 --> 02:35:16,358 HOW THAT FITS WITH WHY IS 3898 02:35:16,358 --> 02:35:17,993 INDUSTRY SO RISK-AVERSE IF THESE 3899 02:35:17,993 --> 02:35:22,631 PEOPLE ARE DOING SO WELL? 3900 02:35:22,631 --> 02:35:23,699 AND HOW DOES THAT WORK? 3901 02:35:23,699 --> 02:35:25,367 IS THERE ANY CONCERN THAT THE 3902 02:35:25,367 --> 02:35:26,668 COMPANIES ARE RISK-AVERSE 3903 02:35:26,668 --> 02:35:27,736 BECAUSE THIS FUNDING IS 3904 02:35:27,736 --> 02:35:32,174 AVAILABLE? 3905 02:35:32,174 --> 02:35:33,509 >> THAT'S A VERY IMPORTANT 3906 02:35:33,509 --> 02:35:35,144 QUESTION AND IT'S EASIER FOR US 3907 02:35:35,144 --> 02:35:36,845 TO CONCEPTUALIZE PARTICULARLY 3908 02:35:36,845 --> 02:35:39,081 FOR THE PEDIATRIC AND NEONATAL 3909 02:35:39,081 --> 02:35:40,616 POPULATION. 3910 02:35:40,616 --> 02:35:47,356 LET ME CALL ON DR. CRUX. 3911 02:35:47,356 --> 02:35:49,825 >> DR. MENSAH, CAN YOU HEAR ME? 3912 02:35:49,825 --> 02:35:51,126 >> YES, WE CAN. 3913 02:35:51,126 --> 02:35:51,894 >> OKAY, GREAT. 3914 02:35:51,894 --> 02:35:52,461 THANK YOU. 3915 02:35:52,461 --> 02:35:55,497 THANK YOU FOR THAT QUESTION. 3916 02:35:55,497 --> 02:35:58,400 SORE THIS RISK-AVERSE NATURE OF 3917 02:35:58,400 --> 02:35:59,802 INVESTORS, AND I WANT TO START 3918 02:35:59,802 --> 02:36:01,870 WITH SAYING THE SUCCESS OF OUR 3919 02:36:01,870 --> 02:36:06,408 SMALL BUSINESS PROGRAM ALMOST 3920 02:36:06,408 --> 02:36:08,577 EXCLUSIVELY DEPENDS ON THE 3921 02:36:08,577 --> 02:36:11,447 COMPANIES THAT -- TO BRING IN 3922 02:36:11,447 --> 02:36:12,781 ADDITIONAL CAPITAL FROM PRIVATE 3923 02:36:12,781 --> 02:36:14,750 SOURCES LIKE INVESTORS AND 3924 02:36:14,750 --> 02:36:18,120 PARTNERS, AND THE FACT THAT WE 3925 02:36:18,120 --> 02:36:19,488 MENTIONED, AS DR. MENSAH 3926 02:36:19,488 --> 02:36:21,323 MENTIONED, INVESTORS ARE 3927 02:36:21,323 --> 02:36:24,393 RISK-AVERSE AND THAT'S ALMOST A 3928 02:36:24,393 --> 02:36:30,566 PLANGT STATABLANKET STATEMENT, T 3929 02:36:30,566 --> 02:36:31,767 FACT, ESPECIALLY BECAUSE THESE 3930 02:36:31,767 --> 02:36:33,001 DAYS, THERE ARE A LOT OF 3931 02:36:33,001 --> 02:36:35,504 TECHNOLOGIES THAT HAVE ADVANCED 3932 02:36:35,504 --> 02:36:38,373 TO LATER STAGES OF DEVELOPMENT, 3933 02:36:38,373 --> 02:36:41,276 SO INVESTORS HAVE A LOT MORE 3934 02:36:41,276 --> 02:36:45,747 OPTIONS, SO THE SMALL BUSINESS 3935 02:36:45,747 --> 02:36:47,049 PROGRAM, BY THE TIME OUR 3936 02:36:47,049 --> 02:36:48,851 COMPANIES FINISH A PHASE TWO 3937 02:36:48,851 --> 02:36:51,053 PROGRAM, THEY ARE NOT AS 3938 02:36:51,053 --> 02:36:52,688 ADVANCED AS MOST INVESTORS LIKE 3939 02:36:52,688 --> 02:36:53,555 THEM TO BE. 3940 02:36:53,555 --> 02:36:55,991 AND THAT'S WHERE THAT RISK 3941 02:36:55,991 --> 02:36:56,992 FACTOR COMES IN. 3942 02:36:56,992 --> 02:36:59,495 SO PROVIDING THIS ADDITIONAL 3943 02:36:59,495 --> 02:37:02,464 PHASE 2B FUNDING ALLOWS OUR 3944 02:37:02,464 --> 02:37:07,636 COMPANIES TO DO THIS NEEDED TO 3945 02:37:07,636 --> 02:37:10,139 CROSS THAT VALLEY OF DEATH, TO 3946 02:37:10,139 --> 02:37:15,310 COMPLETE SOME REALLY CRITICAL 3947 02:37:15,310 --> 02:37:16,311 DE-RISKING ASK YOU STUDIES SO 3948 02:37:16,311 --> 02:37:17,312 THEY CAN GET TO A STAGE WHERE 3949 02:37:17,312 --> 02:37:18,947 THEY CAN ATTRACT INVESTORS AND 3950 02:37:18,947 --> 02:37:22,551 PARTNERS. 3951 02:37:22,551 --> 02:37:27,689 DOES THAT ANSWER THE QUESTION? 3952 02:37:27,689 --> 02:37:29,391 >> I SEE HEAD NODDING, JANE. 3953 02:37:29,391 --> 02:37:31,260 ALL RIGHT. 3954 02:37:31,260 --> 02:37:32,361 IF THERE AREN'T ANY OTHER 3955 02:37:32,361 --> 02:37:33,929 QUESTIONS, IF YOU COULD GO AHEAD 3956 02:37:33,929 --> 02:37:37,633 AND PLEASE RATE THEM SEPARATELY. 3957 02:37:37,633 --> 02:37:47,876 5028 AND 5029. 3958 02:38:18,640 --> 02:38:20,809 >> I THINK WE'RE GOOD TO GO. 3959 02:38:20,809 --> 02:38:21,310 >> THANK YOU. 3960 02:38:21,310 --> 02:38:22,644 WE NOW HAVE THE LAST FIVE 3961 02:38:22,644 --> 02:38:24,379 CONCEPTS FOR YOUR CONSIDERATION. 3962 02:38:24,379 --> 02:38:26,281 THEY ARE ALL PART OF OUR 3963 02:38:26,281 --> 02:38:27,583 CATALYZE SUITE OF PROGRAMS. 3964 02:38:27,583 --> 02:38:29,351 YOU HEARD ABOUT THEM THIS 3965 02:38:29,351 --> 02:38:31,420 MORNING FROM DR. PECK AND ALSO 3966 02:38:31,420 --> 02:38:34,089 FROM DR. GIBBONS' REPORT. 3967 02:38:34,089 --> 02:38:36,358 I'D LIKE TO GIVE CREDIT TO 3968 02:38:36,358 --> 02:38:38,894 DR. MIKE PECK, WHO'S THE PROGRAM 3969 02:38:38,894 --> 02:38:41,630 DIRECTOR FOR THE ENTIRE SUITE OF 3970 02:38:41,630 --> 02:38:44,399 PROGRAMS. 3971 02:38:44,399 --> 02:38:45,801 THE CATALYZE PROGRAM WAS 3972 02:38:45,801 --> 02:38:47,002 LAUNCHED FIVE YEARS AGO TO 3973 02:38:47,002 --> 02:38:48,537 PROVIDE NHLBI WITH A 3974 02:38:48,537 --> 02:38:50,772 COMPREHENSIVE SUITE OF PROGRAMS, 3975 02:38:50,772 --> 02:38:53,175 SUPPORTS, AND SERVICES TO 3976 02:38:53,175 --> 02:38:56,111 EFFICIENTLY AND EFFECTIVELY HELP 3977 02:38:56,111 --> 02:38:59,514 INVESTIGATORS BRING THE HLBS 3978 02:38:59,514 --> 02:39:01,516 RELATED INNOVATIONS FROM THE 3979 02:39:01,516 --> 02:39:04,486 PRODUCT DEFINITION STAGE THROUGH 3980 02:39:04,486 --> 02:39:05,887 RESEARCH AND REGULATORY 3981 02:39:05,887 --> 02:39:06,221 SUBMISSIONS. 3982 02:39:06,221 --> 02:39:08,090 WE ARE ASKING COUNCIL'S SUPPORT 3983 02:39:08,090 --> 02:39:12,694 FOR THE RENEWAL OF FIVE 3984 02:39:12,694 --> 02:39:13,695 UNDERLYING RFAs. 3985 02:39:13,695 --> 02:39:16,131 EACH CONCEPT NEEDS TO BE RATED 3986 02:39:16,131 --> 02:39:20,736 SEPARATELY. 3987 02:39:20,736 --> 02:39:24,239 TO QUOTE JIM KILEY, IT'S NOT AS 3988 02:39:24,239 --> 02:39:25,107 BAD AS IT SOUNDS. 3989 02:39:25,107 --> 02:39:27,109 YOU'RE NOT GOING TO HEAR FIVE 3990 02:39:27,109 --> 02:39:27,776 REPEATED PRESENTATIONS. 3991 02:39:27,776 --> 02:39:29,511 AT THE END, I'LL REMIND YOU WHAT 3992 02:39:29,511 --> 02:39:31,179 THE TITLES ARE TO MAKE IT A 3993 02:39:31,179 --> 02:39:31,847 LITTLE BIT EASIER FOR YOU TO 3994 02:39:31,847 --> 02:39:34,149 RATE THEM SEPARATELY. 3995 02:39:34,149 --> 02:39:37,219 ONE RFA, THE FIRST ONE I'LL TALK 3996 02:39:37,219 --> 02:39:40,222 ABOUT, ADDRESSES THE ENABLING 3997 02:39:40,222 --> 02:39:41,523 TECHNOLOGIES AND TRANSFORMATIVE 3998 02:39:41,523 --> 02:39:43,091 PLATFORMS. 3999 02:39:43,091 --> 02:39:45,294 THAT'S WHEN YOU HAVE THE REALLY 4000 02:39:45,294 --> 02:39:48,997 CRAZY IDEA, THAT'S WHERE YOU GO. 4001 02:39:48,997 --> 02:39:50,766 TWO ADDRESSES SMALL MOLECULES 4002 02:39:50,766 --> 02:39:53,835 AND BIOLOGICS, AND THE LAST TWO 4003 02:39:53,835 --> 02:39:54,636 ADDRESS MEDICAL DEVICE 4004 02:39:54,636 --> 02:39:55,937 PROTOTYPES AND OTHER 4005 02:39:55,937 --> 02:39:57,706 THERAPEUTICS. 4006 02:39:57,706 --> 02:40:00,309 THE GRANTS THAT ARE FUNDED BY 4007 02:40:00,309 --> 02:40:04,179 THESE FIVE RFAs ARE 4008 02:40:04,179 --> 02:40:05,280 SUPPLEMENTED BY TECHNICAL AND 4009 02:40:05,280 --> 02:40:07,549 BUSINESSES SERVICES FROM THE 4010 02:40:07,549 --> 02:40:09,484 CATALYZE COORDINATING CENTER. 4011 02:40:09,484 --> 02:40:11,019 LET ME GIVE A BRIEF DESCRIPTION 4012 02:40:11,019 --> 02:40:16,958 OF WHAT EACH RFA IS DOUGH S DESO 4013 02:40:16,958 --> 02:40:18,393 DO AND THEN SHARE WITH YOU WHAT 4014 02:40:18,393 --> 02:40:20,462 IMPACT THE PROGRAMS HAVE HAD AND 4015 02:40:20,462 --> 02:40:23,065 WHY WE ARE ENTHUSIASTIC ABOUT 4016 02:40:23,065 --> 02:40:25,434 THEM. 4017 02:40:25,434 --> 02:40:27,936 THE CONCEPT 5030 IS A RENEWAL 4018 02:40:27,936 --> 02:40:30,172 NOFO FOR THE ENABLING 4019 02:40:30,172 --> 02:40:31,807 TECHNOLOGIES AND TRANSFORMATIVE 4020 02:40:31,807 --> 02:40:33,475 PLATFORMS THAT WILL SUPPORT THE 4021 02:40:33,475 --> 02:40:34,776 DEVELOPMENT OF THE NEXT 4022 02:40:34,776 --> 02:40:37,212 GENERATION OF PREDICTIVE 4023 02:40:37,212 --> 02:40:39,414 DIAGNOSTIC AND THERAPEUTIC 4024 02:40:39,414 --> 02:40:42,684 PRODUCTS OR MODEL SYSTEMS 4025 02:40:42,684 --> 02:40:44,353 RELEVANT TO THE NHLBI MISSION 4026 02:40:44,353 --> 02:40:46,321 AREAS. 4027 02:40:46,321 --> 02:40:50,992 THAT'S 5030. 4028 02:40:50,992 --> 02:40:52,694 5031 AND 5032 ADDRESS SMALL MOL 4029 02:40:52,694 --> 02:40:54,563 MOL KEULS AND BIOLOGICS AND HERE 4030 02:40:54,563 --> 02:40:57,032 THE FOCUS IS ON TARGET 4031 02:40:57,032 --> 02:40:57,766 IDENTIFICATION AND VALIDATION 4032 02:40:57,766 --> 02:40:59,434 FOR THE FIRST ONE, AND THEN 4033 02:40:59,434 --> 02:41:02,170 PRELIMINARY PRODUCT AND LEAD 4034 02:41:02,170 --> 02:41:03,105 SERIES IDENTIFICATION. 4035 02:41:03,105 --> 02:41:08,243 THE LAST TWO ARE CONCEPTS 4036 02:41:08,243 --> 02:41:09,745 5033 AND 5034, AND THEY ADDRESS 4037 02:41:09,745 --> 02:41:11,747 MEDICAL DEVICE PROTOTYPES 4038 02:41:11,747 --> 02:41:15,283 DEVELOPMENT AND TESTING, AND 4039 02:41:15,283 --> 02:41:19,988 VALIDATION. 4040 02:41:19,988 --> 02:41:20,856 I'LL TELL YOU ABOUT THE IMPACT 4041 02:41:20,856 --> 02:41:21,723 OF THESE PROGRAMS. 4042 02:41:21,723 --> 02:41:23,392 SINCE THE CATALYZE PROGRAM 4043 02:41:23,392 --> 02:41:25,360 LAUNCHED BACK IN FISCAL YEAR 4044 02:41:25,360 --> 02:41:27,562 2019, THE CATALYZE SUITE OF 4045 02:41:27,562 --> 02:41:31,333 NOFOs THAT RESULTED IN 4046 02:41:31,333 --> 02:41:33,735 253 APPLICATIONS AND 51 FUNDED 4047 02:41:33,735 --> 02:41:34,503 AWARDS. 4048 02:41:34,503 --> 02:41:36,938 THE 51 FUNDED AWARDS HAVE 4049 02:41:36,938 --> 02:41:40,575 RESULTED IN 60 PUBLICATIONS, 23 4050 02:41:40,575 --> 02:41:42,711 U.S. PATENT APPLICATIONS, AND 4051 02:41:42,711 --> 02:41:44,913 SIX INTERNATIONAL PATENT 4052 02:41:44,913 --> 02:41:45,747 APPLICATIONS. 4053 02:41:45,747 --> 02:41:47,516 THREE OF THE U.S. PATENTS HAVE 4054 02:41:47,516 --> 02:41:49,251 ALREADY BEEN AWARDED. 4055 02:41:49,251 --> 02:41:52,421 THERE HAVE BEEN SEVERAL FDA FI 4056 02:41:52,421 --> 02:41:55,524 FILINGS FOR PREIND AND Q 4057 02:41:55,524 --> 02:41:55,824 SUBMISSIONS. 4058 02:41:55,824 --> 02:41:58,794 IN ADDITION, OF THE 22 COMPLETED 4059 02:41:58,794 --> 02:42:00,762 PROJECTS WHICH WERE FUNDED IN 4060 02:42:00,762 --> 02:42:04,866 COHORT 1 AND COHORT 2, 2019 AND 4061 02:42:04,866 --> 02:42:06,501 2020, 3 PROJECTS HAVE ALREADY 4062 02:42:06,501 --> 02:42:08,804 ADVANCED TO THE CATALYZE 4063 02:42:08,804 --> 02:42:11,907 PRE-CLINICAL PHASE. 4064 02:42:11,907 --> 02:42:16,144 THREE PROJECTS WERE PROACTIVELY 4065 02:42:16,144 --> 02:42:18,780 TERMINATED BY THE INSTITUTE DUE 4066 02:42:18,780 --> 02:42:23,552 TO TECHNICAL FEASIBILITY ISSUES. 4067 02:42:23,552 --> 02:42:24,820 16 PROJECTS HAVE CONTINUED ALONG 4068 02:42:24,820 --> 02:42:27,656 THEIR PRODUCT DEVELOPMENT 4069 02:42:27,656 --> 02:42:30,025 PATHWAY, AND MOST IMPORTANTLY, 4070 02:42:30,025 --> 02:42:32,961 12 OF THE 16 PROJECTS HAVE 4071 02:42:32,961 --> 02:42:36,798 RECEIVED NEARLY $15 MILLION IN 4072 02:42:36,798 --> 02:42:40,001 FULL-ON FUNDING, INCLUDING FOUR 4073 02:42:40,001 --> 02:42:43,171 ADDITIONAL NHLBI SBIR AWARDS, 4074 02:42:43,171 --> 02:42:44,906 THREE R01 AWARD, AND FUNDING 4075 02:42:44,906 --> 02:42:48,577 FROM OTHER ORGANIZATIONS. 4076 02:42:48,577 --> 02:42:49,978 ONE OF THE GOALS OF THE CATALYZE 4077 02:42:49,978 --> 02:42:52,280 PROGRAM IS TO ATTRACT 4078 02:42:52,280 --> 02:42:53,615 GEOGRAPHICALLY AND 4079 02:42:53,615 --> 02:42:55,016 DEMOGRAPHICALLY DIVERSE 4080 02:42:55,016 --> 02:42:57,152 INNOVATORS FROM BOTH ACADEMIC 4081 02:42:57,152 --> 02:43:01,022 AND FOR-PRO FIPROFIT INSTITUTIO. 4082 02:43:01,022 --> 02:43:02,657 WE'VE ACTUALLY BEEN MODESTLY 4083 02:43:02,657 --> 02:43:04,759 SUCCESSFUL ON THE GEOGRAPHIC 4084 02:43:04,759 --> 02:43:05,193 DIVERSITY FRONT. 4085 02:43:05,193 --> 02:43:06,928 THE PROGRAM HAS RECEIVED 4086 02:43:06,928 --> 02:43:08,830 APPLICATIONS FROM ORGANIZATIONS 4087 02:43:08,830 --> 02:43:11,132 IN 30 U.S. STATES, AND WE'VE 4088 02:43:11,132 --> 02:43:13,335 FUNDED ORGANIZATIONS IN 19 OF 4089 02:43:13,335 --> 02:43:15,337 THESE STATES. 4090 02:43:15,337 --> 02:43:17,138 THE 51 AWARDS REPRESENT A 4091 02:43:17,138 --> 02:43:18,840 DIVERSE MIX OF INSTITUTIONS, 4092 02:43:18,840 --> 02:43:23,311 ABOUT TWO THIRDS OF THEM 4093 02:43:23,311 --> 02:43:28,750 ACADEMIC, 15% SMALL BUSINESSES, 4094 02:43:28,750 --> 02:43:30,485 8% BEING NON-PROFIT RESEARCH 4095 02:43:30,485 --> 02:43:32,120 ORGANIZATIONS. 4096 02:43:32,120 --> 02:43:34,656 I MUST ADMIT WE HAVE MORE WORK 4097 02:43:34,656 --> 02:43:36,658 TO DO TO ADVANCE OUR DIVERSITY 4098 02:43:36,658 --> 02:43:39,594 BY RACE AND ETHNICITY AND ALSO 4099 02:43:39,594 --> 02:43:41,730 DIVERSITY BY GENDER. 4100 02:43:41,730 --> 02:43:43,965 WE ANTICIPATE THAT THE RENEWAL 4101 02:43:43,965 --> 02:43:47,769 OF THE CATALYZE PROGRAM WILL 4102 02:43:47,769 --> 02:43:50,171 CONTINUE TO ATTRACT A DIVERSE 4103 02:43:50,171 --> 02:43:53,742 POOL OF INNOVATORS AND 4104 02:43:53,742 --> 02:43:54,709 NON-FEDERAL COST SHARING AND 4105 02:43:54,709 --> 02:43:57,746 ALSO ENABLE US TO GENERATE MORE 4106 02:43:57,746 --> 02:43:59,214 INTELLECTUAL PROPERTY BUT ALSO 4107 02:43:59,214 --> 02:44:00,982 TO DIVERSIFY THE PROGRAM. 4108 02:44:00,982 --> 02:44:06,021 SO LET ME BRIEFLY SUMMARIZE BY 4109 02:44:06,021 --> 02:44:07,856 TELLING YOU ABOUT -- IF I CAN 4110 02:44:07,856 --> 02:44:13,061 SEE MY CHEAT SHEET HERE. 4111 02:44:13,061 --> 02:44:15,030 SO 5030 IS THE ENABLING 4112 02:44:15,030 --> 02:44:17,499 TECHNOLOGIES PLATFORM. 4113 02:44:17,499 --> 02:44:19,234 5031 IS THE DEFINITION FOR SMALL 4114 02:44:19,234 --> 02:44:21,536 MOLECULES AND BIOLOGICS, THAT'S 4115 02:44:21,536 --> 02:44:23,271 FOR TARGET IDENTIFICATION AND 4116 02:44:23,271 --> 02:44:27,809 VALIDATION. 4117 02:44:27,809 --> 02:44:29,778 5032 IS THE PRODUCT DEFINITION 4118 02:44:29,778 --> 02:44:32,280 FOR SMALL MOLECULES AND 4119 02:44:32,280 --> 02:44:34,482 BIOLOGICS AS A PRELIMINARY AND 4120 02:44:34,482 --> 02:44:38,219 LEAD SERIES IDENTIFICATION, AND 4121 02:44:38,219 --> 02:44:40,789 5033 IS MEDICAL DEVICE PROTOTYPE 4122 02:44:40,789 --> 02:44:41,957 DESIGN AND TESTING. 4123 02:44:41,957 --> 02:44:46,061 AND THEN THE LAST ONE, 5034, IS 4124 02:44:46,061 --> 02:44:48,997 MEDICAL DEVICE PROTOTYPE TESTING 4125 02:44:48,997 --> 02:44:50,365 CHARACTERIZATION AND VALIDATION. 4126 02:44:50,365 --> 02:44:51,232 SO THE WHOLE COMPREHENSIVE 4127 02:44:51,232 --> 02:44:52,100 SUITE. 4128 02:44:52,100 --> 02:44:53,635 THANK YOU VERY MUCH. 4129 02:44:53,635 --> 02:44:55,337 LET ME PAUSE HERE TO SEE IF YOU 4130 02:44:55,337 --> 02:44:58,273 HAVE ANY QUESTIONS AND I KNOW 4131 02:44:58,273 --> 02:44:59,140 DR. PECK IS ONLINE TO HELP ME 4132 02:44:59,140 --> 02:45:02,110 OUT IF THERE ARE QUESTIONS THAT 4133 02:45:02,110 --> 02:45:04,512 I CAN'T ANSWER RIGHT HERE IN 4134 02:45:04,512 --> 02:45:04,746 PERSON. 4135 02:45:04,746 --> 02:45:05,680 >> PLEASE. 4136 02:45:05,680 --> 02:45:09,718 >> SO I REALLY DO LIKE THE 4137 02:45:09,718 --> 02:45:12,354 SUPPORT THAT'S BUILT INTO THESE 4138 02:45:12,354 --> 02:45:17,425 PROGRAMS AND TO HELP THE P.I.s 4139 02:45:17,425 --> 02:45:18,860 AND -- THE PROGRAM. 4140 02:45:18,860 --> 02:45:20,261 MY QUESTION IS EVERY TIME I 4141 02:45:20,261 --> 02:45:22,564 REVIEW GRANTS FOR THESE 4142 02:45:22,564 --> 02:45:24,466 MECHANISMS, THERE ALWAYS SEEMS 4143 02:45:24,466 --> 02:45:27,402 TO BE A VERY LIMITED NUMBER OF 4144 02:45:27,402 --> 02:45:31,239 APPLICATIONS PER CALL, AND I'M 4145 02:45:31,239 --> 02:45:35,243 WONDERING WHETHER, ONE, IS IT 4146 02:45:35,243 --> 02:45:39,948 WORTH FOLDING ALL FIVE INTO ONE 4147 02:45:39,948 --> 02:45:41,282 AND RE-LOOKING AT HOW THAT 4148 02:45:41,282 --> 02:45:42,684 PROGRAM IS STRUCTURED? 4149 02:45:42,684 --> 02:45:45,020 TWO, IS THERE OPPORTUNITY TO 4150 02:45:45,020 --> 02:45:47,322 CONTINUE TO INCREASE THE POOL OF 4151 02:45:47,322 --> 02:45:51,259 APPLICATIONS THAT COME IN, GET 4152 02:45:51,259 --> 02:45:54,162 MORE DIVERSITY, BECAUSE IT'S 4153 02:45:54,162 --> 02:45:56,765 LIKE -- OFTEN IT'S ONE OR THREE 4154 02:45:56,765 --> 02:45:59,934 APPLICATIONS PER FUNDING CYCLE 4155 02:45:59,934 --> 02:46:01,870 APPLYING FOR EACH OF THESE, SO 4156 02:46:01,870 --> 02:46:02,470 COMMENT ON THAT? 4157 02:46:02,470 --> 02:46:05,974 >> I'M GLAD TO SEE MY 4158 02:46:05,974 --> 02:46:07,208 [INAUDIBLE], BUT MY SHORTEST 4159 02:46:07,208 --> 02:46:10,178 ANSWER TO YOU IS YES, DEFINITELY 4160 02:46:10,178 --> 02:46:11,079 VERY THOUGHT-PROVOKING AND WE 4161 02:46:11,079 --> 02:46:14,015 SHOULD CONSIDER ALL OPTIONS. 4162 02:46:14,015 --> 02:46:14,883 WHAT DO YOU THINK? 4163 02:46:14,883 --> 02:46:17,252 >> IN TERMS OF EVALUATION PANEL, 4164 02:46:17,252 --> 02:46:18,820 COME UP WITH THE SAME STATEMENT 4165 02:46:18,820 --> 02:46:20,088 FOR HOW YOU'RE DOING THIS. 4166 02:46:20,088 --> 02:46:21,489 ONE WAY WE'RE TRYING TO ADDRESS 4167 02:46:21,489 --> 02:46:23,191 THIS IS BY IMPROVING OUR 4168 02:46:23,191 --> 02:46:24,492 MARKETING OUTREACH, AND THEN 4169 02:46:24,492 --> 02:46:26,027 ALSO ADDRESSING MAYBE -- THESE 4170 02:46:26,027 --> 02:46:28,163 ARE VERY CHALLENGING, LIKE YOU 4171 02:46:28,163 --> 02:46:30,999 HAVE TO COME IN WITH A -- 4172 02:46:30,999 --> 02:46:32,734 MATCHING FUND -- SO IT'S 4173 02:46:32,734 --> 02:46:35,537 TRACKING THE DIFFERENT NAWD 4174 02:46:35,537 --> 02:46:39,374 NA -- SOAGAIN THERE'S THAT CHALE 4175 02:46:39,374 --> 02:46:42,010 OF -- BUT GREAT QUESTIONS AND I 4176 02:46:42,010 --> 02:46:46,114 THIITHINK WE'LL TAKE THOSE BACK. 4177 02:46:46,114 --> 02:46:47,115 >> THANK YOU. 4178 02:46:47,115 --> 02:46:48,483 ANY OTHER ADDITIONAL QUESTIONS 4179 02:46:48,483 --> 02:46:50,518 OR SUGGESTIONS FOR THE 4180 02:46:50,518 --> 02:47:00,695 INSTITUTE? 4181 02:47:06,234 --> 02:47:06,735 ALL RIGHT. 4182 02:47:06,735 --> 02:47:08,369 THE EASIEST ONE TO REMEMBER IS 4183 02:47:08,369 --> 02:47:09,938 THE CRAZY IDEAS PLATFORM, THE 4184 02:47:09,938 --> 02:47:11,139 FIRST ONE. 4185 02:47:11,139 --> 02:47:12,674 AND THEN MAYBE THINK OF THEM IN 4186 02:47:12,674 --> 02:47:15,076 TERMS OF THE SMALL MOLECULES AND 4187 02:47:15,076 --> 02:47:16,644 BIOLOGICS FOR THE SECOND TWO. 4188 02:47:16,644 --> 02:47:19,514 AND THE MEDICAL DEVICE 4189 02:47:19,514 --> 02:47:19,814 PROTOTYPES. 4190 02:47:19,814 --> 02:47:21,583 AND IN EACH OF THE TWO, THERE'S 4191 02:47:21,583 --> 02:47:23,918 THE EARLY PHASE PRODUCT 4192 02:47:23,918 --> 02:47:26,654 DEFINITION AND THEN THERE'S THE 4193 02:47:26,654 --> 02:47:30,391 LATER PHASE FOR VALIDATION 4194 02:47:30,391 --> 02:47:31,593 AND -- THAT'S HOW I THINK ABOUT 4195 02:47:31,593 --> 02:47:41,669 IT. 4196 02:47:44,272 --> 02:47:44,973 THAT'S A GOOD WAY TO THINK ABOUT 4197 02:47:44,973 --> 02:47:49,577 IT. 4198 02:47:49,577 --> 02:47:51,112 >> THANKS, MIKE. 4199 02:47:51,112 --> 02:47:51,746 >> ALL RIGHT. 4200 02:47:51,746 --> 02:47:53,882 IF YOU COULD GO AHEAD AND 4201 02:47:53,882 --> 02:47:56,618 COMPLETE YOUR RATING. 4202 02:47:56,618 --> 02:48:06,761 PLEASE. 4203 02:49:07,255 --> 02:49:09,457 >> HOW ARE WE DOING ON THE 4204 02:49:09,457 --> 02:49:09,691 VOTING? 4205 02:49:09,691 --> 02:49:11,292 >> WE JUST HAVE THREE MORE 4206 02:49:11,292 --> 02:49:12,227 PEOPLE WE'RE WAITING ON, AND 4207 02:49:12,227 --> 02:49:14,295 THEN I CAN SEE THE SENSITIVITY 4208 02:49:14,295 --> 02:49:15,630 ANALYSIS. 4209 02:49:15,630 --> 02:49:16,664 >> ALL RIGHT. 4210 02:49:16,664 --> 02:49:17,765 WITH THREE MORE TO GO, I'LL TURN 4211 02:49:17,765 --> 02:49:19,934 IT OVER TO DR. PRENGER. 4212 02:49:19,934 --> 02:49:30,278 THANK YOU VERY MUCH. 4213 02:49:31,079 --> 02:49:32,780 >> WHILE YOU FINISH VOTING, WE 4214 02:49:32,780 --> 02:49:33,982 WILL BE ABLE TO TABULATE THE 4215 02:49:33,982 --> 02:49:39,053 RESULTS AND PUT IT UP. 4216 02:49:39,053 --> 02:49:42,523 >> WE'RE ALMOST THERE. 4217 02:49:42,523 --> 02:49:43,758 JUST A FEW VOTES AWAY. 4218 02:49:43,758 --> 02:49:45,093 JUST A FEW RATES AWAY. 4219 02:49:45,093 --> 02:49:55,603 AND I CAN PUT THAT UP FOR YOU. 4220 02:50:04,178 --> 02:50:04,312 OKAY. 4221 02:50:04,312 --> 02:50:05,580 >> WE'LL PUT THOSE UP AFTER WE 4222 02:50:05,580 --> 02:50:06,281 CLOSE THE SESSION. 4223 02:50:06,281 --> 02:50:10,718 >> OKAY. 4224 02:50:10,718 --> 02:50:16,858 EVERYONE'S FINISHED THEN. 4225 02:50:16,858 --> 02:50:17,759 >> OKAY. 4226 02:50:17,759 --> 02:50:19,294 THANK YOU VERY MUCH, EVERYONE. 4227 02:50:19,294 --> 02:50:22,697 THAT CONCLUDES THE OPEN SESSION 4228 02:50:22,697 --> 02:50:26,634 OF OUR COUNCIL TODAY. 4229 02:50:26,634 --> 02:50:28,169 SO DR. GIBBONS, WOULD YOU LIKE 4230 02:50:28,169 --> 02:50:34,475 TO CLOSE US OUT? 4231 02:50:34,475 --> 02:50:38,179 >> THANK YOU FOR YOUR SERVICE. 4232 02:50:38,179 --> 02:50:40,882 WE STAND ADJOURNED. 4233 02:50:40,882 --> 02:50:41,215 GREAT JOB. 4234 02:50:41,215 --> 02:50:43,918 >> THANK YOU VERY MUCH. 4235 02:50:43,918 --> 02:50:54,262 [RECORDING STOPPED]