>> GOOD AFTERNOON.
WELCOME TO THE 188th MEETING
OF THE NICHD ADVISORY COUNCIL.
THANK YOU FOR YOUR PATIENCE AS
WE HAD SOME TECHNICAL
DIFFICULTIES BEFORE WE STARTED.
TODAY'S MEETING IS OPEN TO THE
PUBLIC, BROADCAST ON THE NIH
VIDEOCAST NETWORK.
THANK YOU FOR --
SORRY.
TOMORROW ON DAY 2 WE'LL HOLD OUR
CLOSED SESSION.
I APPRECIATE EVERYONE WHO HAS
JOINED US TODAY BY TEAMS OR ON
THE VIDEOCAST.
WITH THE VIRTUAL MEETING FORMAT
UNMUTE YOURSELF BEFORE SPEAKING
INTO YOUR HOME MICROPHONE.
STATE YOUR NAME SO THE
TRANSCRIBER AND VIDEOCAST
OBSERVERS WILL KNOW WHO IS
SPEAKING.
AFTER YOU SPEAK, PLEASE BE SURE
TO MUTE YOUR MICROPHONE TO
PREVENT ANY FEEDBACK LOOPS OR
OTHER AUDITORY INTERFERENCES.
NOW I WILL TURN THE MEETING OVER
TO DR. REBEKAH RASOOLY.
>> I WANT TO SAY ONE WORD OF
THANKS TO ABOUT TEN TECHNICAL
PEOPLE OFF TO MY RIGHT,
INCLUDING THE STAFF FROM NIH
VIDEOCAST AND OUR MEETING
CONTRACTORS WHO REALLY RESCUED A
DIFFICULT TECHNICAL SITUATION IN
RECORD TIME.
SO LET'S JUST OFFER THEM THANKS.
WE CANNOT HOLD THESE MEETINGS,
WHICH ARE PUBLIC AND OPEN,
UNLESS WE IN FACT MAKE THEM OPEN
TO THE PUBLIC SO THIS WAS A
HEROIC ACT.
ON THE COUNCIL MEMBER WEBSITE,
YOU SHOULD HAVE READ AGREED, TO
SIGNED THE RULES FOR SPECIAL
GOVERNMENT EMPLOYEES BEFORE
EVALUATING NIH -- ANY NIH GRANT
APPLICATIONS.
ALSO, REMEMBER THAT YOU CANNOT
SERVE ON ANY NIH PEER REVIEW
PANEL WHILE SERVING AS A COUNCIL
MEMBER, AND YOU CANNOT PROVIDE
SECOND LEVEL REVIEW AT COUNCIL
FOR ANY APPLICATION IN WHICH YOU
PARTICIPATED IN FIRST LEVEL PEER
REVIEW.
AN INDIVIDUAL CANNOT SERVE ON
BOTH FIRST AND SECOND LEVELS OF
PEER REVIEW.
LET'S MOVE ON TO APPROVAL THE
COUNCIL MINUTES FROM OUR JANUARY
MEETING.
THE MINUTES FOR THE JANUARY 2025
MEETING WERE POSTED ON YOUR
COUNCIL MEMBER WEBSITE FOR YOUR
CONSIDERATION.
I'M GOING TO ASK YOU TO VOTE TO
APPROVE OR NOT APPROVE THESE
MINUTES AND LET ME JUST EXPLAIN
TO YOU HOW THE VOTING WORKS
USING THIS TEAMS PLATFORM.
WE'RE GOING TO ASK FIRST FOR
PEOPLE WHO WANT TO VOTE IN FAVOR
OF THE APPROVAL, AND WE'LL ASK
YOU TO DO THAT BY RAISING YOUR
TEAMS HAND, YOUR VIRTUAL HAND.
AND THEN THE MEETING ORGANIZER
WILL LOWER YOUR HAND.
THEN I WILL CALL FOR PEOPLE WHO
ARE OPPOSED TO APPROVING IT AND
AGAIN PLEASE RAISE YOUR HAND AND
JUST LEAVE IT UP.
AND THEN FINALLY, WE'LL ASK FOR
ANYPLACE WHO WANTS TO ABSTAIN.
AGAIN RAISE YOUR HAND AND THEN
WE'LL LOWER IT AS WE COUNT YOU.
IF YOUR HAND IS NOT RAISED FOR
YES, NO, OR ABSTAIN YOU WILL BE
NOTED AS NOT PRESENT SEW FOR THE
VOTE.
SO NOW LET US MOVE TO CONSIDER
THE JANUARY 2025 COUNCIL
MINUTES.
MAY I HAVE A MOTION TO APPROVE
THE MINUTES?
>> SO MOVED.
>> IS THAT DR. CEDARS?
>> YES.
>> AND A SECOND?
>> SECOND.
>> ALL RIGHT.
AND SINCE THIS IS A VIRTUAL
MEETING EVERYONE SHOULD PLEASE
VOTE.
ALL IN FAVOR RAISE YOUR HAND AND
LEAVE IT UP.
DR. JABS CAN'T RAISE HER HAND IN
TEAMS.
WE'RE WORKING ON ALL THIS.
ONLY MEMBERS SHOULD BE VOTING,
NOT OUR AD HOC MEMBERS.
JUST ONLY MEMBERS OF WHOM WE
HAVE EIGHT I BELIEVE.
OKAY.
ANYONE OPPOSED PLEASE RAISE YOUR
HAND.
FINALLY, IS ANYONE INDICATING
THAT THEY WILL ABSTAIN, PLEASE
RAISE YOUR HAND.
OKAY.
SO, THE MINUTES FROM JANUARY
2025 HAVE BEEN APPROVED.
LET ME JUST GO THROUGH THE
FUTURE MEETING DATES AND THEN
I'LL TURN THE MEETING BACK OVER
TO DR. CERNICH.
WE'RE MEETING NEXT, THIS WILL BE
AN IN-PERSON MEETING IN
SEPTEMBER, ON THE 9th AND
10th.
AND THAT WILL BE ON THE NIH
CAMPUS.
AND THEN IN JANUARY, END OF
JANUARY, 26th AND 27th, FOR
A VIRTUAL MEETING.
THE REST OF THE DATES ARE ALSO
ON YOUR AGENDA.
DR. CERNICH?
>> THANK YOU, REBEKAH.
I WANT TO THANK EVERYBODY FOR
JOINING US TODAY AND THANK YOU
AGAIN FOR BEARING WITH US WITH
THE TECHNICAL DIFFICULTIES.
SO, LET ME START BY INTRODUCING
MYSELF.
I THINK SOME OF YOU KNOW ME
ALREADY.
BUT FOR THOSE WHO DO NOT, I'M
ALISON CERNICH, I HAVE BEEN
DEPUTY DIRECTOR OF NICHD SINCE
2019.
AND I WAS ASKED TO STEP IN AS
ACTING DIRECTOR OF NICHD IN
APRIL AND HAVE BEEN DOING SO
SINCE THEN.
PRIOR TO MY SERVICE AS THE
DEPUTY DIRECTOR OF NICHD, I WAS
DIRECTOR FOR NATIONAL CENTER FOR
MEDICAL REHABILITATION RESEARCH
HERE AT NICHD.
PRIOR TO THAT I WAS AT THE
DEPARTMENT OF VETERANS AFFAIRS
WHERE I HELD MULTIPLE ROLES
INCLUDING LIAISON FOR
PSYCHOLOGICAL HEALTH AND
TRAUMATIC BRAIN INJURY PROGRAM
DEPARTMENT OF DEFENSE.
I HAVE AN INTRAMURAL SCIENCE
PROGRAM, WORKING WITH THE NIH
REHABILITATION MEDICINE
DEPARTMENT AT THE CLINICAL
CENTER, WORKING ON INTEGRATING
DISABILITY FUNCTIONING DATA INTO
THE "ALL OF US" PLATFORM AND
ALSO DEVELOPING ASSISTIVE
TECHNOLOGY MEASURE FOR THAT
PLATFORM WITH THE TEAM THERE.
SO JUST WANT TO LET YOU KNOW A
LITTLE BIT OF BACKGROUND ABOUT
ME.
ALSO I WANT TO SAY THANK YOU TO
DR. BIANCHI FOR HER EXCELLENT AS
MUCH AS AS DIRECTOR UNTIL THIS
YEAR.
I WANT TO TALK ABOUT SOME
PROGRAM UPDATES WE HAVE AT
NICHD.
A NUMBER OF UPDATES RELATED TO
HHS AND NIH LEADERSHIP.
SOME UPDATES RELATED TO THE
BUDGET.
AND THEN PROGRAM AND POLICY
UPDATES.
YOU WILL NOTICE WE'RE LIGHT ON
SCIENCE THIS TIME.
WE'VE HAD A NUMBER OF THINGS
HAPPEN, WE WANT TO MAKE SURE YOU
HAVE THE INFORMATION YOU NEED TO
TRACK WHAT'S HAPPENING HERE.
SO WITH RESPECT TO OUR PROGRAM,
I DO WANT TO HIGHLIGHT AND THANK
COUNCIL FOR THEIR FANTASTIC
INPUT AS WE MOVE THROUGH OUR
STRATEGIC PLAN REFRESH,
PUBLISHED IN JANUARY OF 2025.
AS WE NOTED IN PREVIOUS COUNCILS
THE STRATEGIC PLAN HAS NOT
CHANGED SUBSTANTIVELY IN TERMS
OF OUR OVERARCHING RESEARCH
THEME.
AND RESEARCH GOALS.
WE DID HAVE SOME UPDATES TO THE
ASPIRATIONAL GOALS AND THOSE ARE
NOTED IN THE PLAN.
THIS IS STILL AVAILABLE ON OUR
WEBSITE.
AND I JUST WANT TO NOTE AS WELL
THAT ONE OF THE REASONS THAT WE
REFRESHED INSTEAD OF FULLY
OVERHAULING WAS THAT WE WERE
TRACKING THROUGH IMPLEMENTATION
PLAN A NUMBER OF THE SCIENTIFIC
ADVANCES AND PROGRESS RELATED TO
OUR GOALS AND FELT THAT WE HAD
ANOTHER FIVE YEARS OF WORK TO DO
BUT WE DID ADJUST SOME
OBJECTIVES TO MAKE SURE WE
ACCOUNTED FOR EITHER PROGRESS IN
THE FIELD OR PROGRESS THAT WE
WERE TRACKING WITH RESPECT TO
PROGRAMMATIC UPDATES AND
CHANGES.
SO, THIS IS AVAILABLE ON THE
WEBSITE NOW.
I ALSO JUST WANT TO THANK THE
STAFF WHO DID AN INCREDIBLE JOB.
WE WERE WELL AHEAD OF SCHEDULE
AND GRATEFUL TO GET THIS OUT IN
JANUARY.
AND THANKS SO MUCH TO ALL OF
YOU.
IT WAS A HUGE EFFORT, WHEN I
COUNTED 75 TO 100 STAFF THAT
MADE THIS HAPPEN.
SO, THE OTHER THING I JUST WANT
TO CALL EVERYONE'S ATTENTION TO,
THIS IS, AGAIN, ALSO ON OUR
WEBSITE, WE USUALLY GO THROUGH A
NUMBER OF SCIENTIFIC UPDATES,
BUT GIVEN PROGRAMMATIC AND NIH
AND HHS-WIDE UPDATES WE WANTED
TO GIVE YOU, WE WANTED TO CALL
YOUR ATTENTION TO OUR 2024
RESEARCH HIGHLIGHTS RECAP.
THERE'S SO MANY PLACES WHERE THE
SCIENCE ADVANCED BASED ON
EFFORTS OF THE EXTRAMURAL
COMMUNITY AND INTRAMURAL
COMMUNITY AND REALLY HAS
ADVANCED OUR STRATEGIC PLAN BUT
ALSO SCIENCE IN THESE MAJOR
AREAS.
AND I JUST WANT TO MAKE SURE
FOLKS HAVE THAT AS A PUBLIC
RESOURCE IF THEY WANT TO TALK
ABOUT WHAT NICHD ACCOMPLISHED IN
2024.
ANOTHER UPDATE RELATED TO NICHD
IS THAT MANY OF YOU ARE FAMILIAR
WITH THE DATA AND SPECIMEN HUB,
DASH.
THIS IS A CENTRALIZED RESOURCE
FOR RESEARCHERS TO SHARE
DE-IDENTIFIED DATA FROM STUDIES
FUNDED BY NICHD.
IT SEARCHES AS A PORTAL FOR
REQUESTING BIOSPECIMENS FOR
SELECTED STUDIES IN THERE.
WE HAVE 230 STUDIES ON 60
TOPICS, AND THERE ARE MANY
SPECIMENS AVAILABLE INCLUDING
BLOOD AND BREAST MILK.
WE ARE NOW UNDERGOING A
MIGRATION TO A NEW PLATFORM, AND
DASH IS CURRENTLY UNAVAILABLE.
IT WAS UNAVAILABLE BEGINNING
ABOUT MAY 5.
WE'RE MOVING OVER TO THE
BIOMEDICAL RESEARCH INFORMATICS
COMPUTING SYSTEM, BRICS, HOSTED
BY NIH.
AND THIS IS A PLATFORM THAT WILL
PROVIDE ADDED FUNCTIONALITY, AND
INCREASED SECURITY.
THIS IS A MODULAR WEB-BASED
SYSTEM SO WE WILL ESSENTIALLY BE
AN AN STANCE TO THAT PLATFORM.
ALL THE DATASETS PREVIOUSLY WILL
STILL BE AVAILABLE.
THEY ARE JUST GOING TO BE
MIGRATING, DATA WILL BE
MIGRATING OVER TO THAT PLATFORM.
WE HOPE IT WILL BE BACK
AVAILABLE IN FALL OF 2025.
THE OTHER THING I JUST WANT TO
NOTE THAT WE HAVE BEEN WORKING
ON WITH NIH IS THE AUTISM DATA
SCIENCE INITIATIVE, RELEASED
RECENTLY, BEING LED BY THE
DIVISION OF PROGRAM COORDINATION
PLANNING AND STRATEGIC
INITIATIVES, WHICH I WILL
CONTINUE TO CALL DPCPSI FROM
HERE ON IN BECAUSE IT'S A
MOUTHFUL.
BUT THEY ARE GOING TO BE LOOKING
AT CAUSATION OF AUTISM SPECTRUM
DISORDER AS WELL AS SOME OF THE
DATA RELATED TO THE
EFFECTIVENESS OF INTERVENTIONS
TO ULTIMATELY IMPROVE OUTCOMES.
AND SO THIS IS UTILIZING
INTEGRATED DATA RESOURCES
ALREADY AVAILABLE WITH THE
APPROPRIATE PRIVACY AND
CONFIDENTIALITY PROTECTION.
WE'RE GOING TO BE TRYING TO
IDENTIFY AND ADDRESS GAPS VIA
TARGETED DATA GENERATION SO THIS
IS NOT A LARGE DATA COLLECTION
INITIATIVE BUT REALLY TARGETED
WITHIN THE SCOPE OF WHAT'S
DETAILED IN THE RESEARCH
OPPORTUNITY ANNOUNCEMENTS.
IT WILL SUPPORT ANALYSIS OF
INTEGRATED DATA RESOURCE WITH
LINKAGES THAT THOSE LINKAGES ARE
GOING TO HAVE TO MEET SPECIFIC
REQUIREMENTS THAT WILL BE
REVIEWED.
ALSO, WE'RE GOING TO HAVE A
VENUE TO REPLICATE ANY ANALYSES
THAT ARE CONDUCTED AS A FOCUS OF
THIS INITIATIVE WHICH YOU'LL SEE
IS IN CONCERT WITH THE
PRIORITIES OF THE NEW NIH
DIRECTOR.
AND THERE IS ALSO NOTED IN HERE
COMPLEMENTARY EFFORT TO DEVELOP
A REAL WORLD DATA PLATFORM BUT
THIS IS NOT THAT SPECIFIC
INITIATIVE.
THIS IS A VERY FAST-MOVING
INITIATIVE, DUE DATE JUNE 27.
WE'RE LOOKING TO PROBABLY DO
AWARDS UP TO $5 MILLION, A
24-MONTH TO 36-MONTH PROJECT
PERIOD.
THE OTHER THING I WANT TO MAKE
SURE YOU ALL ARE AWARE OF
RELATED TO PEDIATRIC HEALTH
RESEARCH FUNDED BY NIH, MANY MAY
KNOW THAT NICHD WAS ASKED TO
FUND A NATIONAL ACADEMY OF
SCIENCE, ENGINEERING, AND
MEDICINE STUDY ON THIS.
AND THIS IS A COMMITTEE THAT IS
EXAMINING CURRENT NIH PEDIATRIC
RESEARCH PORTFOLIO AND
STRUCTURE.
WE HAD A NUMBER OF NIH
INSTITUTES AND CENTERS WHO
PARTICIPATED IN WEBINARS
PROVIDING THIS COMMITTEE MORE
INFORMATION ABOUT WHAT WE DO IN
PEDIATRICS ACROSS THE NIH.
NICHD IS A LOCUS FOR MANAGEMENT
OF THIS STUDY.
THERE'S AN OPEN CALL FOR
PERSPECTIVES.
ONE THAT CLOSES ON JUNE 30 FOR
PEDIATRIC PATIENTS AND THEIR
FAMILIES, THE LINK IS THERE FOR
THAT PARTICULAR CALL FOR
PERSPECTIVES.
AND THEN JULY 1 THERE WILL BE
ONE THAT CLOSES FOR PEDIATRIC
HEALTH RESEARCHERS AND THE LINK
IS THERE FOR THAT.
SO WE DO WANT TO ENCOURAGE
COUNCIL AND THOSE WATCHING TO
SPREAD THIS OUT TO YOUR
COMMUNITIES SO THAT NASEM CAN
TAKE IN THAT INPUT AND
INCORPORATE IN THEIR REPORT.
WE ANTICIPATE THE REPORT WILL BE
RELEASED IN JANUARY OF 2026.
SO, LET ME GIVE A COUPLE UPDATES
RELATED TO HHS AND NIH
LEADERSHIP.
SO, FOR HHS, I THINK MANY OF YOU
ARE AWARE WE HAVE A NEW
SECRETARY.
ROBERT F. KENNEDY JR. WAS SWORN
IN FEBRUARY 13, 2025.
AND ON THE SAME DAY THERE WAS
THE ISSUANCE OF EXECUTIVE ORDER
TO ESTABLISH THE PRESIDENT'S
MAKE AMERICA HEALTHY AGAIN
COMMISSION TO INVESTIGATE AND
ADDRESS ROOT CAUSES OF AMERICA'S
ESCALATING HEALTH CRISIS WITH A
FOCUS ON CHILDHOOD CHRONIC
DISEASE.
SO, THIS WAS WITHIN THIS
EXECUTIVE ORDER ALSO THERE WAS A
CALL FOR ASSESSMENT FROM THE
MAHA COMMISSION TO IDENTIFY KEY
DRIVERS BEHIND CHILDHOOD DISEASE
CRISIS WITH FOUR KEY FOCUS AREAS
RECOMMENDED IN THE REPORT THAT
WAS ISSUED ON MAY 22, INCLUDING
POOR DIET, EXPOSURE TO
CHEMICALS, LACK OF PHYSICAL
ACTIVITY AND OVERMEDICALIZATION.
ALSO NUTRITION TRIALS AND
PRECISION TOXICOLOGY.
A PLAN WILL BE DEVELOPED AND
WORKING WITH NIH TO MAKE THAT
HAPPEN.
THE OTHER THING THE COMMUNITY
MAY WANT TO BE AWARE OF
ESPECIALLY RELATED TO SOME OF
THE ISSUES WE'VE DISCUSSED AT
PREVIOUS COUNCILS RELATED TO
INFANT FORMULA THERE IS A NEW
OPERATION STORK SPEED PRIMARILY
LED BY THE FDA, TO ENSURE
SAFETY, RELIABILITY, NUTRITIONAL
ADEQUACY OF THE INFANT FORMULA.
THERE IS ALSO A REQUEST FOR
INFORMATION ON INFANT FORMULA
NUTRIENT REQUIREMENTS, THE LINK
IS THERE.
THE COMMENTS ARE DUE ON THAT BY
SEPTEMBER 11, 2025.
SO THERE HAVE BEEN CHANGES IN
NIH LEADERSHIP SINCE THE LAST
TIME WE'VE MET.
APRIL 21, 2025, DR. JAY
BHATTACHARYA JOINED US AS NEW
NIH DIRECTOR.
HE TOOK OVER THE POSITION FROM
THE ACTING DIRECTOR, DR. MATT
MEMOLI, ALSO PICTURED HERE, WHO
IS NOW THE NIH PRINCIPAL DEPUTY
DIRECTOR.
A NUMBER OF NEW ACTING
DIRECTORS, I'M ONE.
SO ALL THE NAMES ARE LISTED
THERE.
MANY OF THESE FOLKS WERE EITHER
DEPUTY DIRECTORS OR WERE
DIRECTORS IN OTHER PARTS OF THE
NIH, AND SO JUST WANT TO
ACKNOWLEDGE ALL OF THE NEW
LEADERSHIP ACROSS BUT WON'T GO
INTO THEM FOR THE PURPOSE OF
TIME.
SO DR. BHATTACHARYA HAS BEEN
VERY MUCH ON POINT WITH HIS
LEADERSHIP PRIORITIES.
AND THEY ARE LISTED HERE.
HE HAS FIVE MAIN PRIORITIES THAT
INCLUDE FOCUS ON IMPROVING
POPULATION HEALTH, WHICH REALLY
DOES MESH WITH HIS BACKGROUND AS
A POPULATION RESEARCHER.
REPRODUCIBILITY AND RIGOR WHICH
I MENTIONED RELATED TO AUTISM
INITIATIVE, ABOUT
REPRODUCIBILITY OF THE RESEARCH
WE CONDUCT AND SHARING NEGATIVE
FINDINGS.
INNOVATION AND COLLABORATION AND
REALLY TRYING TO PUSH THE
ENVELOPE ON NEW FINDINGS, NOVEL
FINDINGS, IN RESEARCH PORTFOLIO.
RESEARCH SAFETY AND
TRANSPARENCY, MAKING SURE WE
MAINTAIN HIGHEST STANDARDS AND
ADHERE TO STANDARDS.
AND ACADEMIC FREEDOM.
THIS IS REALLY TO SHARE ACADEMIC
DISAGREEMENTS AND MAKE SURE
SCIENTISTS AT NIH HAVE THE
ACADEMIC FREEDOM TO PUBLISH
THEIR SCIENCE IN THE MOST
UNRESTRAINED WAY POSSIBLE.
NOW I'LL GO INTO THE NICHD
BUDGET WHICH I KNOW HAS BEEN
FRONT OF MIND SINCE THE BUDGET
DOCUMENTS ARE BEING RELEASED.
I'LL SAY WITH RESPECT TO OUR FY
25 BUDGET WE DO HAVE OUR FULL FY
25 BUDGET AS OF NOW.
AND OUR PLAN AT THIS MOMENT, AND
I HAVE REASSURED OUR STAFF AND
OTHERS THAT WE REALLY DO INTEND
TO OBLIGATE ALL OF OUR
APPROPRIATED FUNDS IN FY 25.
SO WE DO HAVE SOME CHALLENGES.
I WILL TALK A LITTLE BIT ABOUT
THOSE IN A MOMENT.
THAT'S OUR CURRENT COMMITMENT TO
YOU.
MAY 2, THE PRESIDENT'S SKINNY
BUDGET WAS RELEASED.
THIS INCLUDED FOR THOSE WHO WERE
ABLE TO REVIEW IT, IT IS
AVAILABLE ON THE WHITE HOUSE
WEBSITE, $18 BILLION CUT TO NIH,
CONSOLIDATION OF 27 INSTITUTES
TO 8, A PROPOSAL TO MERGE WITH
THE NATIONAL NATIONAL INSTITUTN
DEAFNESS, AND FOUR INSTITUTES
AND CENTERS PROPOSED FOR
ELIMINATION INCLUDING FOGARTY,
NATIONAL INSTITUTE ON MINORITY
HEALTH, NATIONAL NATIONAL INSTD
COMPLEMENTARY AND INTEGRATIVE
HEALTH.
THE HHS BUDGET BRIEF WAS
RELEASED ON MAY 30, A 40% CUT
FOR COMBINED NICHD-NIDCD
INSTITUTE, AND NEW NAME FOR THAT
INSTITUTE WHICH WAS NATIONAL
INSTITUTE FOR CHILD AND WOMEN'S
HEALTH, SENSORY DISORDERS, AND
COMMUNICATION.
AND THEN FINALLY ON JUNE 5,
THERE WAS RELEASE OF THE NIH
CONGRESSIONAL JUSTIFICATION,
WHICH ADDED ONE MORE PIECE OF
INFORMATION THAT WE HAVE
ADDITIONAL GUIDANCE TO UTILIZE
TO THE GREATEST EXTENT POSSIBLE,
MULTI-YEAR FUNDING FOR FY 25 AND
FY 26, AND SOME SPECIFIC
DIRECTION ON PERSONNEL,
REDUCTION OR SHIFT.
SO LET ME WALK YOU THROUGH A
LITTLE BIT OF WHAT THIS MEANS.
I'LL TALK ABOUT THE MULTI-YEAR
FUNDING, THAT WILL BECOME
IMPORTANT.
WITHIN THE CONGRESSIONAL
JUSTIFICATION, THERE IS A
REQUEST FOR NIH TO USE 50% OF
CURRENT YEAR OF OBLIGATED FUNDS
AND NEXT YEAR OF OBLIGATED
FUNDS, MULTI-YEAR FUNDING.
FOR THOSE WHO ARE NOT FAMILIAR
WITH MULTI-YEAR FUNDING,
MULTI-YEAR FUNDING PROVIDES
FUNDS FOR MULTIPLE YEARS OF A
GRANT AWARD IN ONE YEAR.
SO, THE CONS ARE IT'S EXPENSIVE
IN THE YEAR OF OBLIGATION,
LIMITS TOTAL AWARDS IN THAT FYF
YOU HAVE THE SAME OR LESS MONEY
BUT MORE UP FRONT CAN YOU DO THE
MATH.
YOU GET THE PICTURE.
THE PRO IS THAT IT OFFERS MORE
FLEXIBILITY IN THE OUT YEARS.
WE'VE DONE A QUICK PICTORIAL TO
SHOW YOU WHAT THE DIFFERENCES
ARE.
SO, NORMAL IS ON THE LEFT.
THAT'S OUR ANNUAL FUNDING.
FOR THOSE FAMILIAR WITH THE
PROCESS YOU WOULD GET A SPECIFIC
OBLIGATION PER YEAR AND SO LET'S
SAY 400 OR $500,000 FOR FIVE
YEARS OF THE R01 GRANT.
MULTI-YEAR FUNDING WHAT WOULD
HAPPEN IS THAT YOU WOULD EITHER
GET THE FIRST THREE YEARS OR ALL
FIVE YEARS OF COMPETING FUNDS IN
YEAR ONE, OR WE CAN ALSO ALLOW
FOR MULTI-YEAR FUNDING IN THE
NON-COMPETING PHASE OF A GRANT,
MEANING WHEN YOU'RE COMING IN
FOR THE LAST THREE TO FIVE YEARS
WE CAN PROVIDE UPFRONT FUNDING.
68% OF FUNDS ARE RESERVED FOR
NON-COMPETING AWARDS
WHAT WE'RE THINKING THIS YEAR,
ESPECIALLY THIS YEAR, TO THINK
ABOUT HOW WE CAN PROVIDE FUNDS
TO GRANTS IN YEARS 3 THROUGH 5,
THOUGH THERE'S ALSO THE
REQUIREMENT IN THIS BUDGET THAT
WE PROVIDE FULLY FUNDED GRANTS
WHICH WOULD MEAN VERY TO HAVE
IDENTIFY FOR 50% OF OUR BUDGET
THOSE THAT ARE COMING IN AS
COMPETING AWARDS PROVIDING FULL
FUNDING FOR AWARDS FOR THE FULL
PHASE OF THOSE RESEARCH PROJECT
GRANTS.
SO, THERE HAVE BEEN OTHER RECENT
CHALLENGES I WANT TO MAKE YOU
AWARE OF.
AND THIS IS -- LET ME GO BACK,
ONE MORE POINT FOR
CLARIFICATION.
YOU MAY HAVE ALSO HEARD OF
NO-YEAR FUNDS, THAT'S DIFFERENT
THAN MULTI-YEAR FUNDING.
NO-YEAR FUNDS CAN THE SPENT AT
ANY TIME AND DO NOT EXPIRE.
IN MULTI-YEAR FUNDING LIKE IN
REGULAR GRANTS, EVENTUALLY THOSE
DOLLARS EXPIRE, YOU HAVE TO
SPEND THOSE GRANTS OUT.
NO-YEAR FUNDS DO NOT EXPIRE.
THEY HAVE TO BE -- THEY HAVE TO
BE EVENTUALLY OBLIGATED, THEY
CAN BE RESCINDED.
AND THIS DID HAPPEN TO US WITH
SOME OF THE COVID-19 PANDEMIC
FUNDING THAT WAS EVENTUALLY
RESCINDED, SOME NO-YEAR FUNDS
THAT WENT UNSPENT.
TO MAKE SURE FOLKS MAKE THE
DIFFERENTIATION BETWEEN
MULTI-YEAR FUNDING AND NO-YEAR
FUNDING.
APOLOGIES.
SO, WE HAVE HAD RECENT
CHALLENGES.
I WANT TO ADDRESS THESE UP FRONT
SO THAT YOU ALL ARE AWARE SINCE
THIS HAS BEEN IN THE NEWS, YOU
HAVE QUESTIONS ABOUT IT, WE DID
LOSE A NUMBER OF STAFF IN KEY
FUNCTIONAL AREAS AS A RESULT OF
SOME INITIATIVES ONGOING WITH
RESPECT TO REDUCING OUR
WORKFORCE.
THIS WAS THROUGH THE
PROBATIONARY STAFF, FORMAL
REDUCTION IN FORCE, AND THEN
ALSO FOLK TAKING ADVANTAGE OF
PROGRAMS THAT WERE OFFERED FOR
EARLY RETIREMENT, VOLUNTARY
SEPARATION, AND DEFERRED
RESIGNATION.
AND SO WE DID LOSE STAFF IN KEY
AREAS, INCLUDING FOR EXAMPLE
COMMUNICATIONS, SO YOU MAY HAVE
NOTICED OUR WEBSITE IS NOT AS
DYNAMIC AS IT USED TO BE.
WE HAVE BEEN MAINTAINING ITS
FUNCTIONS AS MUCH AS WE CAN.
SIMILARLY IN SOME OF OUR
LEGISLATION AREAS FOR THOSE IN
THE STAKEHOLDER COMMUNITY.
AND WE'VE BEEN TRYING TO COVER
DOWN AS MUCH AS POSSIBLE.
SOME FUNCTIONS THAT WE HAD MAY
BE EVENTUALLY CENTRALIZED AT NIH
OR HHS BUT RIGHT NOW WHAT WE'RE
DOING IS WORKING AS A COMMUNITY
TO MAKE SURE FUNCTIONS ARE
SUPPORTED.
YOU MAY HAVE OTHER QUESTIONS
ABOUT WORKFORCE REDUCTIONS BUT
TO BE HONEST MANY OF THESE ARE
NOT ABLE TO BE DISCUSSED IN
DETAIL AT THIS POINT BECAUSE OF
ONGOING LITIGATION, SO I REALLY
MAY NOT BE ABLE TO ANSWER
QUESTIONS ON THIS BUT JUST
WANTED TO LET YOU KNOW WHERE WE
ARE.
WE DID HAVE SOME OTHER
CHALLENGES THAT WE'RE NOW
WORKING THROUGH SO WE HAD PAUSES
IN AC ACQUISITION, NOW
RESTARTING.
WE WERE LUCKY TO HAVE OUR
JANUARY COUNCIL.
WE WERE A LITTLE BIT AHEAD THIS
YEAR AND THAT PLAYED TO OUR
ADVANTAGE.
WE DID HAVE PAUSE IN SOME
SCIENTIFIC REVIEWS; THOSE ARE
NOW RESTARTED.
WE'RE ALSO WORKING ON A REQUEST
TO REDUCE OUR NUMBER OF NOTICE
OF FUNDING ANNOUNCEMENTS OR
OPPORTUNITIES, THAT'S SOMETHING
WE'LL TALK ABOUT AS WE MOVE
FORWARD.
IT'S NOT RIGHT NOW AFFECTING THE
CONCEPTS YOU'LL SEE IN FRONT OF
YOU.
WE ALSO HAVE A NEW POLICY
RELATED TO FOREIGN SUBAWARDS
WITH NEED FOR JUSTIFICATIONS,
AND THIS IS REALLY ABOUT THE
FINALIZATION OF A NEW AWARD
STRUCTURE WHICH IS ANTICIPATED,
IN SEPTEMBER OF 2025.
AND SO I CAN TALK ABOUT THAT IN
A LITTLE BIT MORE DETAIL.
WE ALSO HAVE SOME HOLD ON A
AWARDS AND TERMINATIONS.
THESE ARE THINGS WE'VE BEEN
DEALING WITH DYNAMICALLY.
THE STAFF AT NICHD HAS BEEN
ABSOLUTELY FANTASTIC IN THEIR
FLEXIBILITY, DEDICATION, AND
COMMITMENT TO COMMISSION.
TO BE HONEST WITH YOU, THIS
INSTITUTE HAS DONE A TREMENDOUS
JOB IN BEING NIMBLE AND IT IS A
TESTAMENT TO THE STAFF AND
PROFESSIONALS WE HAVE HERE.
SO I DO WANT TO THANK EVERYBODY
FOR THEIR CONTINUED COMMITMENT
TO MISSION.
I'M GOING BACK ONE MORE ON THIS.
TO FOREIGN SUBAWARDS TO ADDRESS
THAT QUICKLY.
THERE IS A TEMPORARY PAUSE WHILE
WE FINALIZE NEW AWARD STRUCTURE.
SO, WE WILL CONTINUE TO FUND
FOREIGN COMPONENTS BUT THEY WILL
BE STRUCTURED IN INDEPENDENT
SUBPROJECTS UNDER PRIME AWARD.
THIS HAS TO DO WITH FISCAL
TRACKING.
DOES NOT APPLY TO FOREIGN
CONSULTANTS OR PURCHASING
SUPPLIES.
THERE'S A GUIDE NOTICE OUT ON
THIS.
WE CAN SEND YOU THE LINK IN THE
CHAT.
BUT WE WILL EFFECTIVE MAY 1,
THIS IS LISTED IN THE GUIDE
NOTICE NOT ISSUE AWARDS TO
DOMESTIC OR FOREIGN AWARDS
INCLUDING SUBAWARD.
WE WILL NOT ACCEPT PRIOR
APPROVAL REQUESTS TO ADD NEW
AWARD.
WE'LL TRY TO NEGOTIATE WHEN NEW
RENEWING, AND WE'LL ALLOW FUNDS
TO BE BUDGETED WHEN WORK CAN BE
PERFORMED DOMESTICALLY AND WE'LL
BE ABLE TO NEGOTIATE BILATERAL
TERMINATIONS IF A PROJECT IS NO
LONGER VIABLE WITHOUT A FOREIGN
SUBAWARD.
WE'RE WORKING FOR THOSE WORKING
ON CLINICAL TRIALS THAT ARE
AFFECTED BY THIS POLICY WE'RE
NOW WORKING WITH OER AND NIH
O.D. HOW TO ADDRESS THOSE AND
HOPE HAVE HAVE A POLICY OUT OR
SOMETHING THAT WE CAN WORK WITH
YOU ALL ON IN THE NEAR FUTURE.
I WANT TO GIVE A COUPLE OF
PROGRAM UPDATES.
THERE IS A NEW OFFICE THAT'S
BEEN ESTABLISHED AGAIN WITHIN
DPCPSI.
AND THIS IS AN INITIATIVE TO
DEVELOP AND EXPAND USE OF
NON-ANIMAL MODELS.
THIS IS THE OFFICE OF RESEARCH
INNOVATION, VALIDATION,
APPLICATION WITHIN THE NIH
OFFICE OF THE DIRECTOR, ORIVA.
IT WILL COORDINATE EFFORTS TO
DEVELOP AND VALIDATE NON-ANIMAL
APPROACHES, ALONE OR IN
COMBINATION, INCLUDING
ORGANOIDS, TISSUE CHIPS, OTHER
IN VITRO SYSTEMS.
COMPUTATIONAL MODELS, AND REAL
WORLD DATA.
THIS IS NOT TO REPLACE ANIMAL
MODELS, NOT RESTRICTING USE OF
ANIMAL MODELS.
THIS IS JUST AN INITIATIVE TO
ADVANCE THESE METHODS AS
ALTERNATIVES.
I WENT THROUGH THIS ALREADY.
I FORGOT.
I APOLOGIZE.
THE LAST ONE IS THE NEW POLICIES
RELATED TO EARLY CAREER
RESEARCHERS, I WANT TO MAKE SURE
EVERYONE IS AWARE BECAUSE YOU
ALL MAY HAVE FOLKS IN YOUR
NETWORK WHERE THEY HAVE BEEN
AFFECTED.
I WANT TO MAKE SURE THESE
POLICIES ARE HIGHLIGHTED FOR
YOU.
SO, NIH HAS GRANTED THE
AUTOMATIC EXTENSION OF
EARLY-STAGE INVESTIGATOR
ELIGIBILITY, ONE, TWO, OR THREE
CYCLES.
THESE POLICIES ARE HERE FOR
EFIs WITHIN TEN YEARS OF
COMPLETING TERMINAL RESEARCH
DEGREE OR END OF POST GRADUATE
CLINICAL TRAINING, NOT COMPETED
FOR NIH RESEARCH AWARDS, THIS IS
AN AUTOMATIC EXTENSION.
THESE FOLKS DO NOT NEED TO TAKE
ACTION OR SUBMIT REQUEST FOR
EXTENSION.
THERE'S EXTENDED ELIGIBILITY FOR
SOME K AWARDS.
IN THIS INSTANCE, IF A MENTORED
CAREER DEVELOPMENT AWARDEE HAD
AN AWARD THAT PREMATURELY ENDED
ON OR AFTER JANUARY 21, 2025,
THIS ALLOWS INDIVIDUALS TO BE
ELIGIBLE TO APPLY FOR NEW
MENTORED CAREER DEVELOPMENT
AWARD WHO MEET ALL OUR NIH
REQUIREMENTS FOR MENTORED CAREER
AWARDS AND THIS WILL ENABLE
COMPLETION OF CAREER DEVELOPMENT
GOALS AND PROGRESSION TOWARDS
RESEARCH INDEPENDENCE.
INVESTIGATORS PREVIOUSLY AWARDED
COMPETING OR NON-COMPETING
CONTINUATION CAREER DEVELOPMENT
AWARDS THAT PREMATURELY ENDED ON
OR AFTER JANUARY, 2025, WHY IS
THAT IMPORTANT?
WHAT WE TELL PEOPLE ONCE YOU'VE
HAD ONE K YOU CAN'T HAVE ANOTHER
K.
IN THIS INSTANCE THAT IS
ACCEPTED.
I SPOKE QUICKLY AND MAYBE BOUGHT
SOME TIME BACK ON THE AGENDA BUT
I'LL OPEN FOR QUESTIONS.
I CAN SEE THE SCREEN NOW AND MAY
BE ABLE TO CALL ON YOU
POTENTIALLY.
MARCELLE?
>> YES, THANK YOU SO MUCH FOR
THAT OVERSIGHT.
I WAS JUST WONDERING IF THERE'S
ANY IDEA HOW MUCH OF AN IMPACT
THIS MULTI-YEAR FUNDING IS GOING
TO HAVE IN THE FIRST SEVERAL
YEARS.
I'M VERY CONCERNED ABOUT THAT,
GIVEN BOTH THE REDUCTION IN
TOTAL AMOUNT OF REVENUE TO THE
INSTITUTE OR INCOME TO THE
INSTITUTE AND THEN THIS PLAYING
DOWN FURTHER THAT THE FUNDING
LEVEL IS GOING TO BE QUITE LOW.
>> GOOD QUESTION.
REMEMBER FIRST THAT THE
PRESIDENT'S BUDGET, BUDGET IN
BRIEF AND CONGRESSIONAL
JUSTIFICATION, THOSE ARE
PROPOSALS, RIGHT?
40% CUT IN BUDGET IS A PROPOSAL
FROM THE PRESIDENT.
THIS IS NOW IN THE CONGRESS FOR
THEIR CONSIDERATION.
AND THEY ARE THE ONES THAT SET
THE APPROPRIATION LEVEL.
SO WITH RESPECT TO THE BUDGET
CUT REMEMBER THAT IS A PROPOSAL.
I THINK THE MULTI-YEAR FUNDING,
YOU ARE CORRECT.
THAT'S WHAT I LAID OUT.
EVEN IN THE SITUATION IN WHICH
WE DO NOT HAVE A BUDGET CUT, IF
WE HAVE TO SPEND MORE IN
MULTI-YEAR FUNDING UP FRONT
THERE WILL BE A NATURAL
REDUCTION IN THE NUMBER OF
AWARDS THAT WE ARE ABLE TO
ISSUE.
THAT'S AS WE LIKE TO SAY THAT'S
JUST MATH, RIGHT?
IF YOU HAVE MORE MONEY GOING OUT
UP FRONT YOU'LL HAVE TO DO LESS
AWARDS.
AND THAT IS REFLECTED IN THE
BUDGET TABLES THAT ARE IN OUR
CONGRESSIONAL JUSTIFICATION TO
SOME EXTENT.
>> DR. JABS HAS A QUESTION.
DO YOU HAVE INFORMATION
REGARDING CHANGES IN INDIRECT
COST RATES?
>> THANK YOU, DR. JABS.
I THINK BOTTOM LINE IS THAT'S,
AGAIN, A PROPOSAL TO MOVE
INDIRECT COST RATE TO 15%,
REFLECTED IN THE NIH
CONGRESSIONAL JUSTIFICATION.
AND, AGAIN, THAT IS SOMETHING
THAT IS ACTUALLY IN STATUTE, THE
INDIRECT COST IS A CALCULATION
IN STATUTE.
SO THAT'S, AGAIN, SOMETHING THAT
WOULD HAVE TO BE PROPOSED BY
CONGRESS OR CHANGED BY CONGRESS
AS I UNDERSTAND IT.
OTHER QUESTIONS?
TRISHA HAS A QUESTION ABOUT
NO-YEAR FUNDS.
TRISHA?
>> YEAH, AM I AUDIBLE NOW?
OH, THANK YOU.
YEAH, OKAY.
SO, JUST FOR CLARIFICATION,
REGARDING NO-YEAR FUNDS, IS THIS
THE IDEA THAT IT'S -- THERE'S
CURRENT YEAR, PRIOR YEAR, AND
THEN THERE IS -- IT HAS TO GET
RETURNED TO THE TREASURY IN YEAR
3, AM I UNDERSTANDING THE WINDOW
CORRECTLY?
>> CURRENTLY WHAT WE DO IN TODAY
TIMES IS THAT WE'RE USING
CURRENT YEAR FUNDS FOR COMPETING
AND NON-COMPETING.
WE DO OFTEN USE MULTI-YEAR
FUNDS, JUST FOR EVERYBODY'S
KNOWLEDGE NICHD HAS BEEN USING
MULTI-YEAR FUNDS FOR SOME
SMALLER AWARDS.
SO R03S, R21, TRYING TO DO MORE
MULTI-YEAR FUNDING OF THOSE
AWARDS TO GIVE OURSELVES SOME OF
THE OUT-YEAR CUSHION I TALKED
ABOUT.
SO IN THAT INSTANCE WE WOULD
ALLOW FOR MULTI-YEAR FUNDING, WE
WOULD GIVE ALL THE MONEY UP
FRONT FOR MULTI-YEAR.
FOR THE INSTANCES WHICH YOU'RE
TALKING ABOUT WHERE WE GET
OUTSIDE OF CURRENT YEAR FUNDING
AND OBLIGATE THE MONEY, LET'S
SAY YOU HAVE A FOUR-YEAR AWARD
OR FIVE-YEAR AWARD AND WE GIVE
YOU ALL THE MONEY IN YEAR ONE.
THERE ARE SOME RESTRICTIONS IN
THE TIME BY WHICH THAT MONEY
EXPIRES.
IT'S USUALLY FIVE YEARS.
AND SO WHAT WE WOULD HAVE TO DO
IN THAT INSTANCE IS MAKE SURE
THAT FOLKS ARE SPENDING DOWN
THEIR BUDGET BECAUSE WE'LL HAVE
LIMITED ABILITY TO GIVE YOU A NO
COST EXTENSION THAT WOULD ALLOW
FOR YOU TO CONTINUE OUT.
I THINK IF WE NEED MORE DETAIL
ON THIS I MIGHT HAVE TO ASK OUR
BUDGET OFFICER, ALEXIS CLARK, TO
HELP ME.
BUT THAT'S GOING TO BE A LITTLE
BIT DIFFERENT BECAUSE IF YOU
GIVE ALL THE MONEY FOR FIVE-YEAR
UP FRONT, THERE WILL BE SOME
ISSUES WITH THE MONEY EXPIRING.
IN THE CASE OF NO-YEAR FUNDING,
RIGHT, THAT'S NOT WHAT WE
USUALLY USE, IT'S USUALLY UNDER
A SPECIFIC AUTHORITY THAT WE
WOULD ALLOW -- BE ALLOWED TO USE
NO-YEAR FUNDING THOSE DOLLARS DO
NOT EXPIRE BUT EVENTUALLY
CONGRESS CAN SAY IF YOU HAVEN'T
UTILIZED THOSE FUNDS WE WILL
RESCIND THEM.
SO, I KNOW IT'S A LOT OF
FISCAL-ESE.
AND I'M STILL TRYING TO MAKE
SURE IT'S ALL STRAIGHT IN MY
HEAD, TO BE HONEST, BECAUSE
THERE'S A LOT OF TERMS BEING
THROWN AROUND BUT WANT TO MAKE
SURE YOU UNDERSTAND THE LANGUAGE
RELATED TO THIS BECAUSE IT IS
CHANGING.
>> DO YOU WANT TO COME OFF MUTE
AND SHARE YOUR COMMENT ABOUT
MULTI-YEAR FUNDS?
>> YEAH, HI.
I JUST WANTED TO COMMENT THAT
THE DoD, WE ONLY USE
MULTI-YEAR FUNDING.
THE NUMBER OF AWARDS IS LESS UP
FRONT THAN WHAT YOU WOULD ALL
NORMALLY BE USED TO, IT DOESN'T
PUT THE PROJECTS AS RISK.
IN FY 25, WE GOT 57% CUT BUT
PROJECTS FUNDED IN FY 24 AND
PRIOR YEARS ARE SAFE.
SO, THAT'S ONE CONSOLATION.
I WANTED TO PUT IT OUT THERE.
AND THE OTHER THING IS FOR THOSE
PROJECTS FUNDED WITH MULTI-YEAR
WE DO HAVE A SMALL NUMBER OF
THOSE THAT HAVE AN OPTION BUILT
IN SO YOU FUND ONLY UP UNTIL A
CERTAIN POINT THAT'S SEVERABLE
IN CASE YOU DON'T GET LATER YEAR
FUNDS BUT COMMITTED FOR A FIVE
YEAR PERIOD BUT THERE MIGHT BE A
SEVERABLE PERIOD SO YOU BUILD IN
AN OPTION SO IN CASE YOU DON'T
HAVE THE FUNDS YOU CAN KIND OF
STEP OUT.
OVER.
>> THANK YOU, GAYLE.
THE OTHER THING, YOU KNOW, WE'VE
BEEN ALSO TALKING ABOUT HOW DO
WE USE FUNDING STRATEGY AS A WAY
TO HELP FOLKS IN TERMS OF EITHER
PHASING AWARDS OR THINKING
THROUGH HOW TO MAKE THINGS A
LITTLE BIT MORE MANAGEABLE.
YOU KNOW, ONE OF THE OTHER
CHALLENGES, I GOT ASKED AT THE
BOARD OF SCIENTIFIC COUNSELORS
ON FRIDAY, WHAT DO WE TELL
YOUNGER TRAINEES WHO MAYBE ARE
GETTING THEIR FIRST AWARD AND
WOULD HAVE TO MANAGE AN AWARD IN
A DIFFERENT FUNDING ENVIRONMENT,
WHAT WOULD WE DO THERE.
ONE THING WE TALKED ABOUT WAS
THINKING THROUGH HOW TO MANAGE
FOR CONTINGENCIES WHERE THE
RESEARCH MIGHT NOT BE GOING THE
WAY YOU WOULD LIKE.
THINKING THROUGH YOUR PROJECT
MANAGEMENT, MAKING SURE YOU'RE
SPENDING DOWN APPROPRIATELY AND
NOT WAITING TO SPEND DOWN UNTIL
THE LAST MINUTE.
AND SO I THINK IN TERMS OF THE
MANAGEMENT OF FUNDS IT'S GOING
TO DEPEND WHETHER OR NOT WE'RE
MORE USING MULTI-YEAR FUNDING SO
I APPRECIATE WHAT GAYLE SAID,
THERE ARE ALSO SOME ADVANTAGES,
AND EFFECTS ON NUMBER AWARDS.
I THINK DR. JABS ASKED ANOTHER
QUESTION, I THINK I SAW IT COME
ACROSS.
>> DR. JABS, DO YOU WANT TO COME
OFF MUTE AND ASK YOUR QUESTION?
>> YEAH, THAT'S FINE.
THANK YOU.
I'M JUST WONDERING THEN IN LIGHT
OF YOUR DISCUSSIONS NO-COST
EXTENSIONS AND CARRY-FORWARD IS
NOT GOING TO BE OPTIONS IN THE
FUTURE?
>> I THINK THAT'S GOING TO
DEPEND, DR. JABS, ON THE LENGTH
OF THE AWARD, MECHANISM FOR THE
AWARD, AND WHEN THE FUNDS ARE
ISSUED.
AND SO IF THIS IS THE MODEL WE
MOVE TO WE MAY INCLUDE SOMETHING
IN THE SEPTEMBER MEETING TO
BETTER EXPLAIN WHAT THE IMPACT
WILL BE IN MORE BROAD TERMS.
RIGHT NOW WE'RE TRYING TO
DETERMINE EXTENT TO WHICH WE
NEED TO USE THIS AND HOW WE NEED
TO USE THIS, SOMETHING WE'RE
WORKING WITH THE OFFICE OF
BUDGET ON.
AND SO I THINK THIS IS SOMETHING
THAT WE MAY HAVE TO FOLLOW BACK
UP WITH YOU ALL ON, ON THE
SPECIFIC DETAILS AS WE SEE THE
IMPLEMENTATION OF THIS ROLLOUT.
>> DR. CEDARS HAS A QUESTION.
>> YES.
YOU HAD MENTIONED IMPACT ON
FIRST-TIME AWARDEES WITH THIS
MULTI-YEAR AND POTENTIALLY
DIFFICULTY MANAGING BUDGET.
I'M ACTUALLY MORE CONCERNED
ABOUT THE RISK TO FIRST-TIME
PEOPLE APPLYING, AND THAT IF IT
IS HARDER TO GET GRANTS THAT
WE'RE AT RISK OF CONTINUING TO
LOSE A GENERATION OF SCIENTISTS
IN THESE AREAS.
>> I ABSOLUTELY TAKE YOUR POINT,
DR. CEDARS.
AND I THINK THAT THE -- THE
THING THAT WE'RE TRYING TO
MANAGE HERE IS MAKING SURE OUR
EARLY STAGE INVESTIGATORS, NEW
INVESTIGATORS, TRAINEES, THIS IS
WHERE THE BALANCE COMES INTO
PLAY, RIGHT?
WE ALSO RECOGNIZE THAT WHEN
SENIOR INVESTIGATORS ARE ALSO
IMPACTED, SOME RESOURCES THEY
RELY ON IN TERMS OF THOSE SENIOR
INVESTIGATOR FUNDS, ALSO HAVE AN
IMPACT.
YOU'RE ALSO TRYING TO LOOK AT
HOW YOU BALANCE THE ECOSYSTEM,
AND IT'S DELICATE AS YOU'RE WELL
AWARE.
IF WE MAKE A CHANGE IN ONE
PLACE, IT'S GOING TO AFFECT
SOMETHING ELSE.
IN THE CONGRESSIONAL
JUSTIFICATION THE BUDGET CUT
WILL IMPACT TRAINING PROGRAMS,
NETWORKS, YOU KNOW, RESEARCH
CENTERS, ET CETERA.
IT WILL NOT CHANGE THE FUNDING
MODEL FOR THOSE NECESSARILY.
SO, THAT IS -- IT'S IN THE
COMPETING RESEARCH PROJECT GRANT
LINE WHERE THE MULTI-YEAR
FUNDING WILL BE AT ISSUE.
SO, I THINK THIS IS WHERE WE'RE
GOING TO WORK TO BALANCE FROM
ECOSYSTEM PERSPECTIVE AND MAKE
SURE WE'RE PROTECTING THE NEXT
GENERATION SO THEY HAVE FUNDS TO
CONTINUE THEIR SCIENCE.
I THINK I SEE JOSE.
DR. CONTRERAS-VIDAL?
>> THANK YOU.
I WOULD LIKE TO ASK FOR A BIT
MORE INFORMATION ABOUT THE NEW
INITIATIVE OUT OF THE NIH
DIRECTOR, NON-ANIMAL MODELING
AND DATA.
>> YES.
THIS IS NOW JUST TAKING SHAPE.
AND SO THE ISSUE HERE IS I THINK
YOU ALL MAY BE FAMILIAR, THERE'S
A COMMON FUND PROGRAM LAUNCHED
BY THE NIH OFFICE OF DIRECTOR
TWO YEARS AGO, COMPLIMENT AIRY,
CO-CHAIRED BY RICK WOYCHIK AND
WORKED WITH THEM, WHAT WE WOULD
LIKE TO DRIVE IS TRANSLATIONAL
HUMAN MODELS.
AND/OR UTILIZATION OF HUMAN DATA
IN EITHER IN CONCERT WITH OR IN
REPLACEMENT OF ANIMAL MODELS.
AND PART OF THIS IS RELATED TO
SOMETHING WE'VE TALKED ABOUT
OVER TIME WITH RESPECT TO I KNOW
WHEN I WAS IN CMRR DIRECTOR, WE
TALKED ABOUT IT, AND HERE AT
COUNCIL, ABILITY TO TRANSLATE
FINDINGS BETWEEN HUMAN AND --
BETWEEN ANIMAL AND HUMAN MODELS,
SO THIS IS ANOTHER WAY TO DRIVE
INNOVATION INTO HUMAN MODELS,
YOU KNOW, ORGANOIDS WE'VE TALKED
ABOUT HERE, MULTIPLE INITIATIVES
ON.
SO THIS IS REALLY SORT OF FROM
THE OFFICE OF THE DIRECTOR A WAY
TO SPUR SOME INITIATIVE, AND
MAKE IT A BIGGER FOCUS OF OUR
PORTFOLIOS.
>> NEW INITIATIVES, ARE THEY
COMING OUT SOON ON THIS PROGRAM?
>> I DON'T KNOW, TO BE QUITE
HONEST.
I CAN FOLLOW UP AND FIND OUT.
>> THANK YOU VERY MUCH.
>> DR. MALDONADO.
>> THANK YOU FOR THE SUMMARY.
I WONDER GIVEN THE FACT THAT YOU
WENT OVER THE STRATEGIC PLAN AND
THAT YOU SAID IT'S FUNDAMENTALLY
NOT VERY DIFFERENT, LOOKING AT
POTENTIAL REDUCTIONS OF THIS
DEGREE AND AGAIN CONCERN ABOUT
NEW INVESTIGATORS.
WILL THERE BE FURTHER GUIDANCE
OR RESTRICTION ON SUBTOPICS OR
THEMES THAT WILL BE BROUGHT UP
FOR CONSIDERATION FOR FUNDING?
ARE WE GOING TO CONTINUE TO HAVE
STUDY SECTION RECOMMENDATIONS AS
THEY EXIST NOW OR WILL THERE BE
THEMES NOT ALLOWED TO BE
ADDRESSED WITHIN THE INSTITUTE
OR MAYBE IF YOU EVEN KNOW WITHIN
CSR AT A WHOLE?
>> IT'S A GOOD QUESTION.
WHAT I CAN TELL YOU IS THIS.
WE'VE NOT BEEN ASKED TO CHANGE
SCIENTIFIC PORTION OF OUR
STRATEGIC PLAN.
AND IN SPEAKING WITH DR.
BHATTACHARYA AND DR. MEMOLI THEY
HAVE NOT RAISED TO ME CONCERNS
ABOUT THE AREAS OUTLINED IN OUR
STRATEGIC PLAN AT THIS POINT.
WITH RESPECT TO CSR AND REFERRAL
GUIDANCE, I DO NOT KNOW THE
ANSWER TO THAT AND WOULD HAVE TO
ASK DR. BURNS AND DR. REID ABOUT
THAT SPECIFICALLY.
RIGHT NOW I DO NOT KNOW OF
ANYTHING -- IF YOU'RE AWARE OFA
PROGRAM TERMINATION IT'S NOT
BEING SOLICITED AT PRESENT.
WE'RE REVIEWING SOME NOTICES OF
FUNDING OPPORTUNITY AND SO SOME
OF THOSE WILL BE REISSUED BUT
THEY MAY BE MODIFIED IN SOME
WAY.
BUT RIGHT NOW FOR NICHD WE HAVE
BEEN WORKING THROUGH OUR NOTICES
OF FUNDING OPPORTUNITY WITH THE
OFFICE OF EXTRAMURAL RESEARCH
WITH DR. LORSCH, AND HIS STAFF,
AS WELL AS OUR INTERNAL STAFF TO
MAKE SURE THAT THEY ARE IN LINE
WITH THE AGENCY PRIORITIES AS
STATED AND THAT WE CAN ACCEPT
THE RESEARCH.
AND SO I THINK FOR RIGHT NOW WE
HAVE NOT TRIED TO RESCOPE
BECAUSE IT'S UNCLEAR WHAT
CHANGES MIGHT BE NEEDED BUT WE
HAVEN'T BEEN GIVEN SPECIFIC
CHANGES RELATE IT'S TO OUR
STRATEGIC PLAN.
CYNTHIA?
>> THANK YOU FOR THE REALLY
THOROUGH AND COMPREHENSIVE
PRESENTATION.
WE APPRECIATE IT.
I RECALL THAT YOU CALLED OUT
AUTISM AS AN AREA THAT WILL BE,
YOU KNOW, UNDER INVESTIGATION IN
SOLICITING APPLICATIONS.
ARE THERE ANY OTHER AREAS YOU
THINK WILL BE HIGHLIGHTED WITHIN
THE SCOPE OF NICHD?
>> SO, I BELIEVE WITH THE MAHA
COMMISSION REPORT CHRONIC
CHILDHOOD DISEASE WILL BE AT
ISSUE.
I'VE TALKED TO DR. BHATTACHARYA
ABOUT DEVELOPMENTAL ORIGINS,
SOME WAYS -- FOR EXAMPLE I KNOW
PRE-TERM BIRTH IS OP OF MIND FOR
YOU.
AND WE HAVE BROUGHT THAT UP
RELATED TO THE AUTISM INITIATIVE
THAT ONE OF THE MAJOR RISK
FACTORS FOR AUTISM IS RELATED TO
PRE-TERM BIRTH AND PARENTAL AGE.
AND SO I THINK FROM OUR
PERSPECTIVE THERE ARE A LOT OF
THINGS THAT ALIGN FROM NICHD'S
RESEARCH PORTFOLIO, ON THE MAKE
AMERICA HEALTHY AGAIN COMMISSION
REPORT.
THERE ARE PLACES WHERE WE THINK
OUR SCIENCE CAN BE A MAJOR
CONTRIBUTOR, IF THERE ARE
FUNDING OPPORTUNITIES THAT ARE
AVAILABLE WE WILL CONTINUE TO
MONITOR AND MAKE SURE NICHD'S
PRIORITIES ARE INCLUDED.
>> WE'RE RIGHT AT TIME.
>> LAST QUESTION TO YOU, DR.
AIZER.
>> RELATED TO THE LAST POINT
MADE IN LOOKING AT THE MAHA
REPORT AND AGENDA IT DOES SEEM,
YOU KNOW, MATERNAL AND CHILD
HEALTH SEEMS TO BE KEY TO THAT
BUT IF I DID MY CALCULATIONS
CORRECTLY, THE TOTAL REDUCTION
IN FUNDS TO THE NEW COMBINED
NICHD IS PRETTY MUCH 40%.
SO THAT DOESN'T SEEM TO REALLY
ALIGN WITH THE PRIORITIES OF THE
MAHA AGENDA, WANT TO KNOW IF YOU
HAVE ANY COMMENTS ON THAT.
>> RIGHT.
THE BUDGET DOCUMENT IS A
PROPOSAL.
WE WON'T KNOW WHAT THE BUDGE SET
UNTIL THAT GOES THROUGH
CONGRESSIONAL APPROVALS BUT I
WILL SAY I THINK YOU ARE CORRECT
THAT THE SCIENCE DOES ALIGN
CLOSELY AND WE WILL CONTINUE TO
WHERE THERE ARE FUNDS AVAILABLE
TO ANSWER THE SCIENCE, CONTINUE
TO MAKE SURE THAT OUR PRIORITIES
ARE REPRESENTED AND WE DID THAT
SPECIFIC TO THE AUTISM
INITIATIVE AND MADE SURE THE
SCIENCE THAT WE KNEW WAS
INCLUDED IN THAT, AS WELL AS
DATA THAT WE HAD AVAILABLE TO
ADDRESS THOSE QUESTIONS, AND I
THINK WE'LL CONTINUE TO DO THE
SAME TO MAKE SURE THAT WE HAVE,
YOU KNOW, THE HISTORY THAT WE
HAVE HERE AT NICHD HAS BEEN A
FOCUS IN THESE AREAS AND WE WILL
CONTINUE TO TRY TO ADVANCE THOSE
PRIORITIES.
ALL RIGHT.
WITH THAT I WILL SIT BACK DOWN.
>> THANK YOU VERY MUCH.
I SEE THAT DR. SEAN MOONEY IS ON
LINE.
AND IT'S A PLEASURE TO INTRODUCE
HIM.
DR. MOONEY IS THE DIRECTOR OF
THE NIH CENTER FOR INFORMATION
TECHNOLOGY, WHERE HE OVERSEES
NIH INFORMATION TECHNOLOGY
SERVICES AND TOOLS.
RANGING FROM COLLABORATION
TECHNOLOGY AND APPLICATIONS FOR
ADMINISTRATORS TO NETWORKS
HOSTING AND TECHNOLOGY TRAINING.
BEFORE JOINING CIT DR. MOONEY
WAS PROFESSOR OF BIOMEDICAL
INFORMATICS AND MEDICAL
EDUCATION AS WELL AS CHIEF
RESEARCH INFORMATION OFFICER AT
UNIVERSITY OF WASHINGTON SCHOOL
OF MEDICINE.
HE WAS ALSO ASSOCIATE DIRECTOR
OF THE NATIONAL ALZHEIMER'S
COORDINATING CENTER, AND
DIRECTOR OF INFORMATICS,
UNIVERSITY OF WASHINGTON
INSTITUTE OF TRANSLATIONAL
HEALTH SCIENCES.
DR. MOONEY GRADUATED FROM
UNIVERSITY OF WISCONSIN-MADISON,
EARNED A Ph.D. IN
PHARMACEUTICAL CHEMISTRY,
UNIVERSITY OF CALIFORNIA, SAN
FRANCISCO, FELLOW OF THE
AMERICAN COLLEGE OF MEDICAL
INFORMATICS, IT'S A PLEASURE TO
INTRODUCE HIM.
WELCOME, DR. MOONEY.
>> HI.
CAN YOU HEAR ME OKAY?
>> WE CAN.
THANK YOU, SEAN.
>> WONDERFUL.
I'M GOING TO SHARE MY SCREEN.
SECOND QUESTION, CAN YOU SEE MY
SCREEN OKAY?
>> WE CAN.
I WANT TO MAKE SURE, IS OUR TEAM
GOOD?
OKAY.
YES, WE'RE GOOD, SEAN.
GO AHEAD.
>> EXCELLENT.
SO FIRST OF ALL, IT'S A HUGE
HONOR TO BE PRESENTING TO YOU
TODAY.
I'M VERY PLEASED TO GET THE
INVITE AND HAPPY TO BE HERE.
I'M SORRY THAT I'M DOING THIS
VIRTUALLY.
WORK TRAVEL CAME UP THAT WAS
SHIFTED BY A WEEK-AND-A-HALF
FORWARD INTO JUNE AND
UNFORTUNATELY OVERLAPPED.
I WAS PLANNING ON GIVING THIS IN
PERSON.
SO I'M SORRY I'M NOT THERE IN
PERSON.
I'M REALLY, REALLY HAPPY TO BE
PRESENTING TODAY.
I'VE BEEN AT THE NIH NOW FOR
ABOUT 15 MONTHS.
AND IT HAS BEEN -- I CAME FROM
THE ACADEMIC WORLD AS YOU HEARD,
AND IT'S BEEN A HUGE WHIRLWIND
TO ABSORB ALL OF THE AMAZING
WORK THAT'S BEEN GOING ON
NIH-WIDE AND ONE OF THOSE THINGS
THAT I'VE LEARNED IS PART OF
THAT PROCESS, IS THAT NICHD HAS
BEEN A LEADER IN DATA, DATA
SHARING, BUILDING THE
INFRASTRUCTURE THAT'S GOING TO
SUPPORT THE DATA, YOU KNOW,
TECHNOLOGIES IN THE FUTURE TO
ENABLE THE SCIENCE THAT WE WANT
TO DO.
DATA SCIENCE IS REALLY KEY AS
YOU ALL KNOW, IN EVERYTHING WE
DO.
AND IN MY ROLE SERVING
TECHNOLOGY AT THE NIH, I'M
REALLY TRYING TO PUT TOGETHER
THE PIECES THAT WILL HELP
SUPPORT THE FUTURE OF RESEARCH
FOR ALL OF THE INSTITUTES, SO
THAT WE CAN BUILD DATA RESOURCES
THAT ARE EFFICIENT,
INTEROPEARABLE, THAT USE
TECHNOLOGY STANDARDS THAT ARE
OFF THE SHELF, YOU KNOW,
HOPEFULLY OPEN, AND SUPPORT ALL
OF THE THINGS THAT WE WANT TO DO
BOTH IN THE INTRAMURAL WORLD BUT
ALSO THE EXTRAMURAL WORLD.
AND SO WE'RE WORKING AND HAVE
PARTNERED WITH A NUMBER OF
INDIVIDUALS AND UNITS ACROSS THE
NIH TO MAKE THIS HAPPEN.
I WANT TO SHOUT OUT REBEKAH
ROSEN AND VALERIE COTTON, VERY
CLOSE PARTNERS WITH THE WORK
WE'VE BEEN DOING AND THAT'S BEEN
JUST REALLY WONDERFUL IN TERMS
OF HOW WE HAVE BEEN BUILDING OUT
WHAT WE WANT TO DO.
AGAIN, AT 15 MONTHS HERE I STILL
FEEL LIKE I'M JUST GETTING
STARTED SO I'M GOING WALK
THROUGH AND TELL YOU ABOUT HOW
WE DO TECHNOLOGY AT THE NIH, HOW
WE ARE BUILDING AND REVISING
WHAT WE DO.
I'LL TALK ABOUT SOME OF THE
SUCCESSES OF INFORMATION
TECHNOLOGY, AND I'LL THEN TALK
ABOUT HOW WE'RE BUILDING
HOPEFULLY THE ECOSYSTEM OF CYBER
INFRASTRUCTURE.
THOSE ARE TECHNOLOGIES THAT
SERVE YOU AND SERVE SCIENTISTS
TO BE ABLE TO ENABLE SCIENTISTS
TO WHAT THEY DO.
I THINK ONE OF THE KEY ELEMENTS
HERE WE DON'T REALLY DEFINE THE
SCIENCE.
I'M A SCIENTIST.
I HAVE A LAB AT NHGRI, I STILL
DO SCIENCE.
BUT WE TRY HARD TO SERVE AND TO
REALLY HELP THE SCIENCE DEFINE
WHAT THE TECHNOLOGIES ARE NEEDED
TO BE PUT IN PLACE THAT COULD BE
MOST RESPONSIVE TO WHAT WE DO.
I'M GOING TO START WITH
COMPUTING AT THE NIH, HOW WE DO
IT.
I'LL TALK ABOUT SOME OPERATIONAL
SUCCESS STORIES TO GIVE AN IDEA
OF THE SCOPE, SCALE AND SUCCESS
OF WHAT WE'VE DONE.
I'LL TALK ABOUT THE CLOUD.
I'LL TALK ABOUT OPERATIONAL A.I.
AND USE OF A.I. IN BIOMEDICINE
AT NIH AND TALK ABOUT BIOMEDICAL
CYBER INFRASTRUCTURE.
I ONLY HAVE 30 MINUTES SO I'LL
GO QUICKLY THROUGH THE MATERIALS
THAT I HAVE TO PRESENT.
WE START WHEN WE THINK ABOUT
TECHNOLOGY AT THE NIH, I THINK
FIRST ABOUT PEOPLE.
THE NIH HAS MORE THAN 6,000
INDIVIDUALS WHO SPEND FULL-TIME
WORK DOING TECHNOLOGY OR I.T.,
I.T.-RELATED WORK AT THE NIH.
AND ALL OF THEM CONTRIBUTE IN
VARIOUS AREAS FROM SUPPORTING,
FOR EXAMPLE, TEAMS MEETING ALL
THE WAY TO SUPPORTING OUR
NETWORK, HIGH PERFORMANCE
CLOUDING, CLOUD INFRASTRUCTURE,
ET CETERA.
ONE OF THE REASONS THE NIH HAS
BEEN SO SUCCESSFUL AT THE
TECHNOLOGIES THAT WE'VE BUILT IS
BECAUSE OF THOSE PEOPLE AND THE
FACT THAT EVERYBODY IS DRIVEN BY
THE NIH'S MISSION TO DO WHAT WE
DO.
WE'VE BUILT INFRASTRUCTURE AND
CYBERSECURITY UPON THAT.
I'LL TALK ABOUT THAT HERE IN A
SECOND.
ENTERPRISE INFRASTRUCTURE
SUPPORTS EVERYBODY.
IT'S THE BIG THINGS.
IT MIGHT BE TO A SCIENTIST THE
BORING THINGS LIKE OUR NETWORK,
ACCESS TO E-MAIL, OUR USE OF,
FOR EXAMPLE, TEAMS AND LICENSES
FOR SOFTWARE THAT WE HAVE.
AND OUR DATA CENTER
INFRASTRUCTURE, THINGS LIKE
THAT.
AND THEN ABOVE THAT THE FEDERAL
GOVERNMENT HAS BEEN INVESTING
HEAVILY IN THE CLOUD AND HOW DO
WE BUILD, YOU KNOW, THIS
TRADITIONAL FIRST HARDWARE INTO
THE CLOUD AND THINKING ABOUT
WHAT BENEFIT THE CLOUD CAN
PROVIDE LIKE AS A SERVICE, ET
CETERA.
THAT MAKES ME THINK ABOUT CYBER
INFRASTRUCTURE, WHAT ARE THOSE
MODULAR TOOLS THAT CAN BE USED
TO CONTRIBUTE TO THE DIGITAL
ECOSYSTEM THAT SCIENTISTS ARE
DEFINING TO SUPPORT THE NIH.
ABOVE THAT WE HAVE DATA, DATA
PLATFORMS, ANALYTICS, AND A.I.
BOTH IN THE SCIENTIFIC WORKED
BUT ALSO ADMINISTRATIVE ONE IN
TERMS OF PERFORMING AND ENABLING
THE WORK THE NIH DOES.
TO TELL YOU ABOUT ENTERPRISE
I.T. AT THE NIH, I WAS ASKED BY
THE DIRECTOR OF THE NIH TO SERVE
AS ASSOCIATE DIRECTOR FOR I.T.
CYBER INFRASTRUCTURE AND
CYBERSECURITY ABOUT A YEAR AGO.
WITHIN THAT ROLE, I OVERSEE THE
OFFICE OF THE CHIEF INFORMATION
OFFICER.
WE RECRUITED A CIO, ADELE
MERITT, OUTSTANDING IN GETTING
HER FEET ON THE GROUND, STARTED
IN SEPTEMBER, A RICH RESEARCH
AND FEDERAL BACKGROUND.
SHE HAS A LOT OF EXPERIENCE TO
REALLY MAKE OCIO SHINE.
OCIO FOCUSES ON I.T.
STRATEGICALLY, ENTERPRISE
ARCHITECTURE, I.T. GOVERNANCE,
INFORMATION SECURITY.
AND ALSO SUPPORTS THE OFFICE OF
THE CHIEF INFORMATION SECURITY
OFFICER.
OPERATIONALLY, I'M ALSO DIRECTOR
OF THE CENTER FOR INFORMATION
TECHNOLOGY, THAT'S ONE OF THE 27
INSTITUTES AND CENTERS OF THE
NIH.
WE PROVIDE ENTERPRISE I.T.
SERVICES BACK TO THE NIH.
REALLY WE'RE THE OPERATIONAL ARM
OF WHAT THE NIH DOES.
WE'RE IN TOTAL ABOUT, I DON'T
KNOW, 1200 OR MAYBE 1300 STAFF
THAT SUPPORT ALL OF THE KIND OF
ENTERPRISE TECHNOLOGIES THAT
SUPPORT WHAT WE DO.
SO, AGAIN, IT'S A BIG
ENTERPRISE, AND OCIO AND CIT
WORK TOGETHER TO PROVIDE BOTH
OUR STRATEGY AS WELL AS OUR
OPERATIONS FOR SUPPORTING WHAT
WE DO.
SO, ALL I.C.s HAVE TECHNOLOGY,
I.T., STRATEGIC STRATEGIES
AROUND DATA AND HAVE INVESTED
HEAVILY IN DATA AND DATA
SCIENCE.
WE HAVE A NUMBER OF PARTNERSHIPS
THAT ENABLE THE TECHNOLOGY SIDE
TO SUPPORT THE BUILDING OF THIS
SO-CALLED CYBER INFRASTRUCTURE
DIGITAL ECOSYSTEM AT THE NIH.
WE WORK WITH THE NATIONAL
LIBRARY OF MEDICINE, WE WORK
CLOSELY WITH THE OFFICE OF DATA
SCIENCE STRATEGY, SUSAN
GREGURICK'S OFFICE.
AND WE PARTNER WITH MANY OF THE
INSTITUTES SUCH AS NICHD IN
TERMS OF HELPING DRIVE US,
TELLING US WHAT THOSE
REQUIREMENTS ARE AND WHAT THE
TOOLS ARE THAT WE CAN PUT IN
PLACE TO HELP SUPPORT THEM.
SO LET ME TALK BRIEFLY ABOUT
SOME OF THE SUCCESS STORIES OF
THINGS WE'VE BUILT AT THE NIH.
SOME OF THESE MIGHT BE BORING TO
SCIENTISTS BUT I'M PROUD OF THE
WORK WE'VE DONE AND IT GOES TO
SHOW THE SIZE AND SCALE OF WHAT
WE DO HERE WITHIN THIS
ORGANIZATION.
THE NIH HAS 45,000 STAFF,
700,000 VISITORS, 200 NIH
BUILDINGS ACROSS NINE GEOGRAPHIC
LOCATIONS.
WE BUILD AND SUPPORT AN NIH
NETWORK FOR SECURE AND RELIABLE
INFORMATION SHARING ACROSS THAT,
SUPPORTING ALL THOSE STAFF AND
VISITORS.
IT'S MORE THAN 4,300 MILES OF
CONNECTED NETWORK CABLING FOR
HIGH-SPEED COMMUNICATIONS AND
DATA TRANSFER ACROSS NIH SITES.
WE ALSO SUPPORT NIH
COLLABORATION TOOLS LIKE ZOOM
AND TEAMS.
AND WE HAVE SUPPORTED MORE THAN
2.5 MILLION VIRTUAL MEETINGS
APPROXIMATELY PER YEAR, A
BILLIONE MAILS RECEIVED AND
SENT, MORE THAN A MILLION GIG A
POINTS IN SHARE POINT AND
ONEDRIVE.
WE SUPPORT BIOWULF, AVAILABLE
WITHIN THE INTRAMURAL PROGRAM.
IT'S IN THE B-12 DATA CENTER,
AND SEES MORE THAN A MILLION CPU
HOURS PER MONTH, SATURATED FROM
USE, BASICALLY RUNNING ALL THE
TIME AND HAS NO OPEN
AVAILABILITY, AND HAS BEEN VERY
POPULAR.
WE SUPPORTED A NUMBER OF
TECHNOLOGIES TO SUPPORT MOVING
RESEARCH AND OPERATIONS TO THE
CLOUD THROUGH THE STRIDES
PROGRAM.
THE STRIDES INITIATIVE IS OUR
PARTNERSHIP WITH AWS, GOOGLE
CLOUD, MICROSOFT AZURE, BUILDING
TECHNOLOGIES ALIGNED WITH NIH'S
STRATEGIC PLAN FOR DATA SCIENCE,
WE WANT TO BUILD MODERN
INTEGRATED, INTUITIVE AND SECURE
TECHNOLOGIES, BRINGING COST
SAVINGS TO THE NIH, SCIENTISTS
OF THE NIH, AS WELL AS TO THE
EXTRAMURAL COMMUNITY THROUGH
STRIDES, AND THROUGH THE STRIDES
DISCOUNTS, AND WE HAVE STRONG
PARTNERSHIPS FOR R&D
COLLABORATIVE ENGAGEMENTS WITH
THOSE PROVIDERS WHO CAN HELP US
ADOPT, YOU KNOW, THE LATEST AND
GREATEST IN TERMS OF WHAT WE DO.
TODAY THERE ARE MORE THAN 364
MILLION GIGABYTES OF DATA IN
STRIDES.
BASICALLY EVERY TYPE OF DATA YOU
WOULD IMAGINE, MOLECULAR, REAL
WORLD CLINICAL DATA, ESSENTIALLY
EVERYTHING THAT MIGHT BE USEFUL
IN BIOMEDICAL IS SOMEWHERE
WITHIN THE STRIDES ORGANIZATION.
WE'VE ONBOARDED 2700 RESEARCH
PROGRAMS, AND HAVE TRAINED MORE
THAN 5500 INVESTIGATION ON USE
OF STRIDES.
STRIDES IS A WONDERFUL
PARTNERSHIP WITH SUSAN
GREGURICK'S OFFICE, OFFICE OF
DATA SCIENCE STRATEGY, AND WE
ARE INCREDIBLY PROUD TO BE PART
OF THE PARTNERSHIP SERVING DR.
GREGURICK'S OFFICE.
MANY NIH AND NIH-FUNDED RESEARCH
PROGRAMS ARE IN STRIDES.
MANY OF THE ONES YOU'VE HEARD
INCLUDING NICHD PROGRAMS, AND
REALLY STRIDES GIVES US THAT
PLATFORM TO BUILD UPON, THAT
HOPEFULLY WE CAN USE TO LEVERAGE
AND BUILD MORE INTEROPERABILITY,
MAKE DATA MORE FAIR, BUT WE WANT
TO BE MORE EFFICIENT AND MORE,
YOU KNOW, MAYBE PLUG AND PLAY IN
TERMS OF HOW WE DO TECHNOLOGIES
TO ENABLE NEXT GENERATION OF
BIOMEDICAL COMPUTING AND ENABLE
THINGS LIKE ADVANCED EMERGENT
TECHNOLOGIES, LIKE ARTIFICIAL
INTELLIGENCE, GENERATIVE A.I.,
LARGE LANGUAGE MODELS, THAT SORT
OF THING.
WORKING WITH NIGMS AND ODSS, WE
HAVE HELPED SUPPORT NIH CLOUD
LAB, WHICH IS PROBABLY MY
FAVORITE -- ONE OF MY FAVORITES
THINGS ABOUT STRIDES.
STRIDES AT ONE SIDE CAN BE
CONSIDERED A DISCOUNT OR ACCESS
TO CLOUD RESOURCES, BUT ON THE
OTHER SIDE IT'S THAT CENTRAL
PLATFORM WHERE WE CAN BUILD,
MORE THAN THE SUM OF ITS PARTS.
AND CLOUD LAB IS ONE OF THOSE
WAYS TO GET INTO THAT.
ANY INVESTIGATOR ASSOCIATED WITH
AN NIH RESEARCH PROGRAM CAN FILL
OUT A FORM, GET $500 OF FREE
CREDIT, ACCESS TO TAILORED CLOD
TRAININGS.
AGAIN, WORKING WITH NIGMS AND
ODSS, THE CLOUD TRAININGS WERE
DEVELOPED BY IDeA STATES
CTRs THAT COLLABORATED TO
CREATE COLLABORATIVE TRAINING.
SO WE'VE TAKEN HERE BASICALLY A
CLOUD DISCOUNT, DISCOUNTED
ACCESS PROGRAM, AND TURNED THAT
INTO COLLABORATIVE TRAININGS
THAT WERE BUILT UPON IT BY
EXTRAMURALLY FUNDED SITES ACROSS
THE COUNTRY.
I THINK THIS IS REALLY A COOL
USE CASE.
AND EXAM EXEMPLIFIES TO SUPPORT
SCIENCE BETTER.
DATA IS KEY, BOTH WITHIN NICHD
STRATEGY BUT ALSO WITHIN
BASICALLY ALL OF BIOMEDICINE AND
IT'S REALLY CRITICAL IN TERMS OF
WHAT WE DO.
WE'RE TRYING VERY, VERY HARD TO
WORK WITH INSTITUTES LIKE THIS
ONE TO HELP DRIVE BUILDING
EFFICIENT, EASY-TO-USE
OFF-THE-SHELF TECHNOLOGIES THAT
CAN MAKE IT -- YOU KNOW, MAKE
SCIENCE BETTER, MAKE SCIENCE
EASIER.
AND HAVE MORE FOCUS ON SCIENCE
AND HOPEFULLY LESS FOCUS ON THE
TECHNOLOGIES.
AND SO WE'RE VERY INTERESTED IN
DEVELOPING AND SUPPORTING
STANDARD PLATFORMS TO SUPPORT
EVERYWHERE FROM DATA, DATA TO
MANAGING DATA, TO DEVELOPING
A.I. MODELS, AND TO ENABLE MODEL
AND DATA SHARING WITHIN AMONGST
RESEARCHERS.
THIS IS A SLIDE, IT'S OUT OF
DATE, COURTESY OF SUSAN
GREGURICK.
AND HER OFFICE.
AND JUST EMPHASIZING THE
INVESTMENTS THE NIH HAS MADE IN
ARTIFICIAL INTELLIGENCE HAVE
BEEN GROWING STEADILY UP TO --
THIS GOES UP TO THE END OF 2023.
I NEED AN UPDATED VERSION OF THE
SLIDE BUT MORE THAN $300 MILLION
A YEAR BASICALLY TWO YEARS AGO,
AND THIS HAS CONTINUED TO GROW.
I THINK OBVIOUSLY A.I. IS
EVERYWHERE.
IT'S NOT TOTALLY CLEAR WHERE
IT'S GOING.
I THINK THERE ARE LOTS OF
OPPORTUNITIES FOR RESEARCH TO
SUPPORT IT AND THE TECHNOLOGY
THAT WE PROVIDE REALLY DRIVES US
TO HELP SUPPORT THIS AS WELL AS
WE CAN.
THERE ARE A NUMBER OF PLATFORMS
THAT EMERGED WITHIN THE CLOUD
THAT HELPED MANAGE DATA TO HELP
DATA WORKFLOWS AND HELP GIVE
PEOPLE ENVIRONMENTS THAT SUPPORT
THE THINGS RESEARCHERS NEED TO
DO, FROM DATA SHARING TO
ANALYSIS, TO BRINGING DATA
TOGETHER, TO STORING AND
MANAGING AND DOING ANALYTICS ON
SPECIFIC DATA, DOING SCIENTIFIC
COMPUTING ON DATA.
YOU MAY HAVE HEARD OF SOME
PLATFORMS OUT THERE, THERE ARE A
NUMBER OF GEN 3, TERRA, CAVATIA,
FOUNDRY, BRICS PLATFORM THAT WE
SUPPORT.
AND ARE HELPING INVESTIGATORS
ADOPT THESE COMMON PLATFORMS,
TRYING TO HELP MAKE THEM
INTEROPEARABLE AND MORE EASY TO
USE.
AND THROUGH THAT WE'RE HOPING
THAT WE CAN BUILD TOOLS MORE
STANDARDIZED TO MAKE THEM MORE
INTEROPEARABLE AND NEED MORE
ENTERPRISE THINKING HOW WE THINK
ABOUT THE DIGITAL ECOSYSTEM OF
TOOLS THAT WE PROVIDE.
WE WANT TO TAKE STANDARD-BASED
APPROACHES TO DO THE WORK THAT
WE DO.
ONE GREAT EXAMPLE, A GREAT
PARTNERSHIP THAT WE HAVE WITH
NICHD, REBEKAH ROSEN HAS BEEN
KEY HELPING DEVELOP THIS, NIH
RESEARCHER AUTH SERVICE, RAS, A
COMMON TOOL TO LOG IN AND
AUTHENTICATE.
THE SYSTEM HAS TO KNOW YOUR
IDENTITY AND TRUST YOU WHEN YOU
COME IN SECURELY.
WE NEED TO ENABLE AUTHORIZATION,
SYSTEM NEEDS TO KNOW WHAT DATA
OR THINGS WITHIN THE SYSTEM YOU
ARE SECURELY AUTHORIZED TO SEE
OR HAVE ACCESS TO OR DO THINGS
WITH.
RAS IS AN OPEN PLATFORM THAT
SOLVES THIS PROBLEM BY ENABLING
RESEARCHERS TO HOPEFULLY NOT
REINVENT THE WHEEL BUT HAVE A
COMMON TOOL KIT FOR ENABLING
BOTH AUTHENTICATION BUT ALSO
AUTHORIZATION USING STANDARDS
THAT ENABLE LIKE ESSENTIALLY
ACCESS, AUTHORIZING ACCESS FOR
INDIVIDUAL WHO IS TRUSTED THAT
HAS THE RIGHTS TO DO IT ACROSS
MULTIPLE RESOURCES.
AND, AGAIN, THIS IS DRIVEN AND
FUNDED BY ODSS, CIT IS PROVIDE
TO BRING THE TECHNOLOGY, AND
NICHD HAS BEEN A KEY LEADER THAT
HAVE REALLY SPEARHEADED THE
IMPLEMENTATION AND DRIVING OF
RAS, SO REALLY PROUD OF THIS
PARTNERSHIP WITH YOU.
RAS IS A LEGO, I THINK OF LEGO
BLOCKS MODULAR, OPEN BLOCKS THAT
CONTRIBUTE TO GLOBAL BIOMEDICAL
CYBER INFRASTRUCTURE SO WE DON'T
HAVE TO KEEP REINVENTING THE
WHEEL EVERY TIME WE WANT TO
INCREASE OR IMPLEMENT NEW
FUNCTIONALITY.
WHAT'S COMING IN THE FUTURE?
SOME THINGS YOU'VE ALREADY SEEN
OR HEARD OF.
WE'RE INTERESTED IN WORKSPACES
THAT ARE IN THE CLOUD THAT
ENABLE DATA INTEGRATION, ENABLE
ANALYSIS OF DATA, OR USE OF A.I.
ON DATA IN A SECURE WAY.
WHEN I WAS AT THE UNIVERSITY OF
WASHINGTON, I WORKED WITH A
NUMBER OF KIND OF CLINICAL DATA
COORDINATING CENTERS COLLECTING
LIKE GENOMICS DATA BUT ALSO REAL
WORLD EHR DATA.
YOU KNOW, RESEARCH CASE
REPORT-LIKE DATA, REDCap DATA,
ET CETERA.
AND THEY WERE IN THE PROCESS OR
MIGRATED TO THE CLOUD, TO THE
STRIDES PROGRAM, FOR ALMOST ALL
OF THEM.
ONE OF THE CHALLENGES THAT THEY
HAD WAS THEY WANTED TO CREATE
INVESTIGATOR SANDBOXES, A WAY
FOR THEIR INVESTIGATORS TO GET
ACCESS IN THEIR ENVIRONMENT IN A
WAY EASY TO SPIN UP AND
ENVIRONMENT, SECURE, LOW COST,
SO WE'RE VERY INTERESTING IN
ENABLING THE SO-CALLED
WORKSPACES IN STANDARDIZED WAY
TO SPIN UP AND ENVIRONMENT AND
CONNECT IT TO DATA USING
TECHNOLOGIES LIKE RAS WHICH I
DESCRIBED IN THE PREVIOUS SLIDE
SUCH THAT WE DON'T HAVE TO
REINVENT THE WHEEL FOR EVERY
RESOURCE WE WANT TO DO.
THE HOPE IS THAT IN THE FUTURE
WE CAN HAVE, YOU KNOW, IN THE
FUTURE WE'LL HAVE A VERY
HOPEFULLY EASY TO USE, I DON'T
KNOW IF I WANT TO USE THE WORD
"VERY" BUT PLUG AND PLAY
ARCHITECTURE TO SPIN UP WHATEVER
THE NEEDS ARE FOR DOING ANALYSIS
FOR DATA THAT HAS BEEN PUT INTO
THE CLOUD IN SOME STANDARDIZED
WAY.
AGAIN, ALL OF THESE TECHNOLOGIES
ARE SERVANT TO THE STRATEGIES
AND NEEDS OF THE I.C.s IN
GENERAL AND, AGAIN, NICHD HAS
BEEN REALLY A LEADER HELPING
DRIVE WHAT WE'VE DONE AND WE'RE
GOING TO CONTINUE TO LOOK TO YOU
FOR MORE LEADERSHIP IN TERMS OF
HOW WE CAN MAKE THIS WORK SUCH
THAT THE FUTURE IS EASIER TO USE
TECHNOLOGIES, AND BARRIERS ARE
LOWER SO MORE OF THESE
STANDARDIZED TECHNOLOGIES ARE
ADOPTED.
A PLOT OF PROGRAMS UTILIZE
WORKSPACES TODAY, AS YOU CAN
SEE.
AND THERE ARE A LOT OF EFFORTS
THAT WE ARE HELPING TO DO TO
HELP SUPPORT THEM.
SOMETHING ELSE THAT'S
INTERESTING THAT I REALLY LIKE
AS WELL THAT WE'RE THINKING A
LOT ABOUT IS THAT A.I. WON THE
NOBEL PRIZE IN CHEMISTRY THIS
YEAR, TWO NOBEL PRIZES ACTUALLY,
AND DAVID BAKER AND OTHERS WHO
WON THE PRIZE IN CHEMISTRY FOR
PROTEIN STRUCTURE PREDICTION.
ONE THING THAT WASN'T READILY
APPARENT WAS THAT UNDERNEATH IT
THE REASON WE KNEW THAT THEY HAD
SOLVED THIS PROBLEM WAS BECAUSE
OF A COMMUNITY OF SCIENTISTS
THAT GOT TOGETHER IN THE EARLY
1990s AND CREATED A
COMPETITION CALLED CRITICAL
ASSESSMENT OF STRUCTURE
PREDICTION, CASP, SCIENTISTS
EVALUATED THEIR METHODS, SOLVING
THE PROTEIN STRUCTURE PREDICTION
PROBLEM.
WITHOUT CASP I WOULD ARGUE THE
FIELD WOULD NOT HAVE ADVANCED
AND WE WOULDN'T KNOW THE PROBLEM
WAS SOLVED.
I'M A HUGE FAN HOW COMMUNITIES
COME TOGETHER IN THE WORLD OF
A.I. AND DATA SCIENCE AND RUN OR
MANAGE THESE SO-CALLED
CHALLENGES WHICH SOUND LIKE
COMPETITIONS BUT ARE MORE THAN
BENCHMARKING OF METHODS OR
COMPETING AGAINST EACH OTHER FOR
METHODS.
THEY ARE REALLY ABOUT
COMMUNITIES COLLABORATING AND
WORKING TOGETHER TO SHOW, TO
IDENTIFY WHAT'S WORKING IN AN
AREA, WHAT IS MAYBE NOT WORKING,
HOW DO WE ADVANCE A FIELD.
CASP IS A GREAT EXAMPLE OVER
35-YEAR SET OF EXPERIMENTS THAT
HAPPENED EVERY TWO YEARS THAT
ENABLED US TO SAY, YEAH, WE'VE
SOLVED THIS PROBLEM.
I'M A HUGE FAN OF CRITICAL
ASSESSMENTS AND DATA CHALLENGES,
EXPECT TO HEAR MORE FROM US
ABOUT THIS, HOW CAN WE ENABLE,
YOU KNOW, UPON ALL THE
TECHNOLOGIES THAT WE'VE BEEN
TALKING ABOUT HOW CAN WE ENABLE
THIS, ENABLE MORE DATA
SCIENCE-LIKE CHALLENGES IN A
STANDARDIZED WAY TO MAKE
ADVANTAGEMENTS HOPEFULLY HAVE
THE TECHNOLOGY AVAILABLE SO
RESEARCHERS CAN MAKE THE
ADVANCEMENTS LIKE THE CASP WORLD
DID WITH PROTEIN STRUCTURE
PREDICTION.
WE HAVE LOTS OF A.I. USE CASES
IN THE OPERATIONAL WORLD AT THE
NIH.
FOR EXAMPLE, HERE'S AN EXAMPLE
OF A BUSINESS CASE OF USING A
GENERATIVE A.I. TOOL, TRIALGPT
THAT ENABLES RESEARCHERS TO
MATCH PATIENTS TO PARTICULAR
CLINICAL TRIALS USING LARGE
LANGUAGE MODELS, OPERATIONAL AND
USED WE'RE HAD HE NIH.
THIS LAST LED TO DEVELOPMENT OF
A NUMBER OF NIH KIND OF
OPERATIONAL A.I. USE CASES.
ONE OF THE THINGS THAT I STILL
TALK ABOUT, I THINK RESEARCHERS
HAVE LEARNED, RESEARCH, THE
RESEARCH WORLD, ME AND MY
COLLEAGUES HAVE LEARNED THIS
NOW, I THINK, BUT WE'VE ENTERED
A NEW AGE, THE AGE OF WHAT I
CALL OPERATIONALLY WHERE WE ARE
HAVING A.I. LIKE GENERATIVE A.I.
METHODS IMPLEMENTED WITHOUT A
DATA SCIENCE ANYWHERE TO BE
FOUND, BASICALLY AN ADVANCED
I.T. SYSTEM THAT'S BEING
IMPLEMENTED.
COULD BE DRAFTING IN-BOX
MESSAGES IN EPIC FOR PROVIDERS
WHEN THEY GET A MESSAGE THROUGH
MY CHART FROM A PATIENT AND
DRAFTING THEIR RESPONSE TO
AMBIENT SCRIBES IN THE CLINIC,
ACCESS TO ENTERPRISE VERSIONS OF
ChatGPT, TO HAVE A CHATBOT.
ALL OF THESE ARE OPERATIONAL
USES OF A.I. THAT ARE
IMPLEMENTED AS ADVANCED I.T.
SYSTEMS.
AND SO I WANT TO EMPHASIZE WE'VE
ENTERED THIS NEW ERA OF
OPERATIONAL A.I. AND RESEARCH IN
GENERAL WE NEED TO THINK VERY
CAREFULLY HOW CAN WE SHIFT AND
REALLY ADAPT TO THIS NEW WORLD
OF A.I. BEING BASICALLY
AVAILABLE EVERYWHERE, INTEGRATED
INTO DIFFERENT PARTS OF OUR
LIVES AND HOW CAN WE ENSURE
PARTICULARLY BIOMEDICINE SPACE
A.I. IS EFFECTIVE, LEGAL,
ETHICAL, RESPONSIBLE, EQUITABLE.
THERE ARE LOTS OF A.I. BENEFITS
TO USE IN THE BUSINESS.
I DON'T NEED TO OVEREMPHASIZE
BUT THERE ARE ALSO RISKS ABOUT
A.I. CAN BE INEQUITABLE, A.I.
CAN ALLOW ESSENTIAL ACTIVE --
SENSITIVE DATA TO BE SHARED AND
TRAIN THE MODEL, THAT MIGHT
EXPOSE SENSITIVE DATA TO THE
COMPANY THAT MADE THE A.I. AND
OTHER USERS OF THAT A.I., CAN
IMPACT HUMAN SAFETY, CAN IMPACT
HUMAN RIGHTS, AND CAN DRIFT IN
ACCURACY.
THERE'S LOTS OF ISSUES WITH
IMPLEMENTATION.
WE'VE BEEN SPEND TIME BUILDING
GOVERNANCE TO SUPPORT OVERSIGHT
FOR OPERATIONAL USES.
I'M GOING TO GO THROUGH THIS
BRIEFLY SINCE I DON'T HAVE A TON
OF TIME LEFT.
ONE OF THE THINGS WE'RE THINKING
ABOUT IS HOW DO WE BUILD THE
WORKFLOW TO MANAGE THE
IMPLEMENTATION OF A.I. AS FAST
AND RESPONSIBLY AS WE CAN.
TODAY THERE'S ACTUALLY -- WE
REPORT TO HHS ANNUALLY ALL OF
OUR A.I. USE CASES, MORE THAN
195 OPERATIONAL USES HAPPENING
AT THE NIH RIGHT NOW, AND 77 OF
THEM ARE ACTUALLY ACTIVE TODAY.
ANOTHER EXAMPLE IS THE
GENERATIVE A.I. SYSTEM THAT'S
USED TO CLASSIFY DATA MANAGEMENT
AND SHARING PLANS TO HELP
PROGRAM OFFICERS CLUSTER AND
SORT THEM.
WE ALSO HAVE RECENTLY HELPED
SUPPORT THE RELEASE OF AN NIH
SECURE GENERATIVE A.I. CHATBOT
USING ENTERPRISE ChatGPT
SUITABLE FOR USE IN THE FEDERAL
GOVERNMENT, IN FED CLOUD, AND
WE'VE GIVEN ALL NIH
INVESTIGATORS ACCESS TO THIS.
WHEN WE RELEASED CHHIRP FOR
ACCESS, WE DID AN INTRODUCTORY
SEMINAR TO INTRODUCE, WITH MORE
THAN 600 PEOPLE FROM THE NIH
JOINED TO HEAR ABOUT GAINING
ACCESS TO A GENERATEIVE A.I.
CHAT BOUGHT SUITABLE FOR SHARING
NIH CONFIDENTIAL INFORMATION.
WE'RE INTRODUCING CO-PILOT, IN
UNIVERSITIES SIMILAR TO
IMPLEMENTS YOU'VE SEEN ALSO AT
YOUR SITE.
I CAN FINISH AND HAPPY TO TAKE
QUESTIONS IF THERE'S TIME OR
WE'RE ALLOWED.
I WANT TO BRING THIS TOGETHER TO
THINK ABOUT WHAT ARE THE TOOLS,
CYBER INFRASTRUCTURE
TECHNOLOGIES THAT WE CAN BUILD
THAT CAN BE BUILT ONCE, USED
MANY TIMES TO ENABLE ALL OF THE
COMPONENTS OF A DATA AND DATA
SCIENCE STRATEGY.
I REALLY THINK ABOUT THIS
DIGITAL ECOSYSTEM.
I LOVE TO THINK ABOUT IT LIKE
LEGO BLOCKS, VERY EASY TO
UNDERSTAND.
WE WANT TOOLS TO BE OPEN, TO BE
REUSABLE, AND INTEROPERABLE SO
YOU CAN THINK ABOUT WHAT PROBLEM
YOU WANT TO SOLVE, TAKE
SOMETHING OFF THE SHELF AND PUT
IT TOGETHER AND GROW AND SCALE
IT FROM THERE.
AND SO THERE'S LOTS OF TOOLS
THAT YOU COULD IMAGINE THINKING
ABOUT.
YOU KNOW, TOOLS THAT GENERATE
DATA, AND SUPPORTING THE
PLATFORMS THAT DO THIS, FROM
BIOINFORMATICS WORKFLOWS TO
SIMPLE THINGS LIKE
AUTHORIZATION, AUTHENTICATION
USE CASE WITH RAS THAT I TALKED
ABOUT EARLIER.
I DEVELOPED A LIST OF TEN SETS
OF PRIORITIES IN THE WORK THAT I
DO, WHAT WE'RE TRYING TO THINK
ABOUT, FLOATING ABOUT, LIKE WHAT
CAN WE SOLVE IN TERMS OF I.T.
THAT WE MIGHT GO AFTER.
WE'VE GROUPED INTO TWO GROUPS,
THINGS THAT HAPPEN WITHIN THE
NIH AND THINGS THAT WE CAN DO
THAT HAPPEN POTENTIALLY BROADLY
WITH THE RESEARCH COMMUNITY AT
LARGE.
WITHIN THE NIH WE'RE INTERESTED
IN ADVANCING HELPING BUILD
TECHNOLOGY TO SUPPORT CLINICAL
RESEARCH AND TRIALS, SUPPORT THE
NIH DATA STRATEGY, SUPPORT
TECHNOLOGY CAREERS AT THE NIH.
WE INVEST IN A.I., WE NEED TO
INVEST IN I.T.
AND SUPPORT USE OF OPERATIONAL
I.T. AT THE NIH AND ENABLE
ENTERPRISE SYSTEMS WITHIN THE
NIH IN THE CLOUD THROUGH THE
STRIDES PROGRAM.
THE BROADER FOCUS OF THE
BIOMEDICAL RESEARCH COMMUNITY
AGAIN THINGS WE'RE FLOATING,
LOOKING FOR FEEDBACK.
ONE IS REALLY CONVENING AND
LEADING BIOMEDICAL SCIENCES
TECHNOLOGY COMMUNITY, TECHNOLOGY
I.I
STS WHO PROVIDE
SOLUTIONS AND LEAD AND ENGAGE,
GIVE THEM A FORUM TO WORK
TOGETHER TO SOLVE THESE MODULAR
PROBLEMS THAT WE HAVE THAT
OFTENTIMES WE SOLVE IN PARALLEL.
WE WANT TO ADVANCE THE
BIOMEDICAL WORK FORCE, TECH
FLUENCY, AND HELP MAKE BETTER
DECISIONS WITH USE OF
TECHNOLOGY.
WE WANT TO SUPPORT SERVICES IN
THE CLOUD ECOSYSTEM AND SUPPORT
NIH CORE RESEARCH FACILITIES
GENERATING DATA AND HAVE
PROBLEMS SHARING DATA AND
MANAGING THE DATA, ARCHIVING
DATA, ET CETERA.
THAT'S ANOTHER AREA WE'RE
THINKING ABOUT.
WE HAVE A BROAD FORTHCOMING
NEWSLETTER, YOU CAN SEE IT ON MY
LINKED IN, OR FRONT PAGE OF CIT.
WILL THAT -- WITH THAT I WILL
FINISH AND THANK YOU FOR THE
OPPORTUNITY, A HUGE HONOR TO BE
HERE, AND I LOOK FORWARD TO
WORKING WITH NICHD IN THE
FUTURE.
YOU'VE BEEN GREAT LEADERS AND
PARTNERS.
>> THANKS, SEAN, FOR JOINING US
TODAY.
THAT WAS A WHIRLWIND TOUR OF
WHAT YOU AND ADELE AND SO MANY,
IVOR AND YOUR COLLEAGUES HAVE
BEEN UP TO.
WE HAVE ANOTHER SPEAKER BUT I
WILL INVITE FOLKS IF THEY WOULD
LIKE IN THE CHAT TO ASK YOU A
COUPLE QUESTIONS THERE BECAUSE
WE DO HAVE DR. KEN MANDL TO TALK
ABOUT PRACTICAL PIECES OF HEALTH
INFORMATICS HERE BUT, SEAN, I
WANT TO THANK YOU FOR YOUR
PARTNERSHIP.
IT'S BEEN TREMENDOUS TO BUILD
OUT SOME OF THE CAPABILITIES AND
SUPPORT THAT YOU'VE GIVEN TO OUR
SYSTEMS IS INCREDIBLE.
THANK YOU FOR JOINING US TODAY.
>> GREAT, THANK YOU.
>> WITH THAT I'LL SAY IT'S A
PLEASURE TO INTRODUCE DR. KEN
KEN
MANDL, DIRECTING A COMPUTATIONAL
HEALTH INFORMATICS PROGRAM AT
BOSTON CHILDREN'S HOSPITAL, AND
LINDBERG PROFESSOR OF PEDIATRICS
AT HARVARD MEDICAL SCHOOL,
TRAINED AS PEDIATRICIAN,
PEDIATRIC EMERGENCY PHYSICIAN, A
MEMBER OF THE NATIONAL ACADEMY
OF MEDICINE, PART OF ITS
LEADERSHIP CONSORTIUM.
DR. MANDL ADVISED TWO DIRECTORS
OF THE U.S. CENTERS FOR DISEASE
CONTROL AND CHAIRING BOARD OF
SCIENTIFIC COUNSELORS NATIONAL
LIBRARY OF MEDICINE.
THANK YOU FOR AGREEING TO
PARTICIPATE AND PRESENT.
>> IT'S AN HONOR TO BE HERE.
AND I'M REALLY PARTICULARLY
APPRECIATING HAVING JUST HEARD
SEAN'S, AS YOU SAID, WHIRLWIND
TOUR.
AND I HAD THE PLEASURE TO KNOW
SEAN AND BE FAMILIAR WITH HIS
WORK.
IT'S AMAZING WHAT HE'S
ACCOMPLISHED FOR THE NIH IN SUCH
A SHORT TIME.
I WOULDN'T SAY MY TALK IS
COMPLEMENTARY WITH HIS.
BUT RIFFING WITH MANY OF THE
THEMES SEAN BROUGHT UP.
YOU MENTIONED IN MY BIO NATIONAL
ACADEMY OF MEDICINE DIGITAL
HEALTH ACTION COLLABORATIVE, AND
A.I. COLLABORATIVE, IT'S COMING
OUT WITH PETER LEE OF MICROSOFT
HEALTH, AND COMING OUT WITH A
DIGITAL ARCHITECTURE VISION
SHORTLY.
THAT'S SOMETHING TO KEEP AN EYE
OUT FOR, PROBABLY THIS SUMMER OR
EARLY FALL THAT WILL ECHO SOME
OF THE THEMES THAT SEAN AND I
ARE PRESENTING.
SO, FOR PEDIATRIC RESEARCH,
THAT'S A BROAD TOPIC.
I'M GOING TO FOCUS TO START WITH
ON A COUPLE OF SORT OF
DIAGNOSTIC USE CASES WITHIN
PEDIATRICS TO ILLUSTRATE HOW WE
MIGHT THINK ABOUT BRINGING
TECHNOLOGY IN SUPPORT OF THAT
WORK ON THE RESEARCH SIDE,
PUBLIC HEALTH SIDE, ALSO
CLINICAL SIDE.
MY OWN SON IS VERY EARLY -- HAD
VERY EARLY ONSET INFLAMMATORY
BOWEL DISEASE, FIRST MANIFESTS
WHEN HE WAS 2 YEARS OLD.
RIGHT NOW HE'S STANDUP COMEDIAN
LIVING IN NEW YORK CITY, DOING
VERY NICELY, SO I'LL CUT TO THAT
CHASE, BUT IT'S BEEN A JOURNEY.
AND I WANT TO FOCUS IN ON ONE
MOMENT IN THAT JOURNEY WHICH IS
WHEN SUDDENLY IN ADDITION TO
HAVING THIS INFLAMMATORY BOWEL
DISEASE, COLITIS, HE ALSO HAD A
SEIZURE DISORDER.
AND I WENT TO HIS PEDIATRIC
NEUROLOGIST WHO WAS THE TOP
SEIZURE DOCTOR IN PROBABLY THE
WORLD, AND THAT DOCTOR, I SAID
COULD THE SEIZURES AND COLITIS
BE RELATED TO EACH OTHER?
AND THE ANSWER WAS UNEQUIVOCALLY
NO.
AND I ASKED THE
GASTROENTEROLOGIST, THE ANSWER
WAS NO.
I HAD THIS SECRET WEAPON, TWO
SECRET WEAPONS.
ONE WAS DATA.
THE SECOND WAS POSTDOCTORAL
FELLOW.
AND WITH THE DATA AND THE
POSTDOCTORAL FELLOW WE LOOKED IN
LARGE DATABASES, INCLUDING OUR
VERY OWN DATABASE AT MY
HOSPITAL, TO SEE WHETHER THIS
RELATIONSHIP EXISTED.
AND WHEN WE STARTED TO COMPARE
THE RELATIONSHIP BETWEEN
SEIZURES AND COLITIS IN THIS ONE
CONDITION, SEIZURES AND COLITIS,
WE DECIDED TO BROADEN AND LOOK
ACROSS ALL AUTOIMMUNE
CONDITIONS.
AND TURNS OUT THAT ACROSS ALL
AUTOIMMUNE CONDITIONS, THE
INCIDENCE OF SEIZURES IS
ELEVATED.
SO THERE ARE IN SOME WAY
CONNECTED MECHANISTICALLY,
PERHAPS GENETICALLY, PERHAPS
THROUGH SOME INSULT, PERHAPS
THROUGH THE MICROBIOME, PERHAPS
THROUGH BRAIN-GUT ACCESS, BUT
THEY ARE CONNECTED.
AND BECAUSE TWO OF THE TOP
CLINICIANS IN THEIR FIELDS
COULDN'T SEE THIS, COULDN'T SORT
OF FOLLOW THIS IN THEIR OWN
EXPERIENCE, WHAT WE HAVE TO
REMEMBER IS THAT THERE ARE
PATTERNS THAT ARE MUCH LARGER
THAN ANYTHING THE MOST
EXPERIENCED CLINICAL BRAINS CAN
DISCERN, PURELY FROM CLINICAL
EXPERIENCE.
AND LARGE SCALE ACCESS TO DATA
AND CAPTURE OF PATIENTS'
EXPERIENCE, MULTI-MODAL DATA, IS
WHAT'S GOING TO MAKE SHE'S --
WHAT'S GOING TO ELUCIDATE THESE
TYPES OF RELATIONSHIPS.
SO, IF YOU -- AND WE ENDED UP
PUBLISHING THIS PAPER IN JAMA
NEUROLOGY, EARLY IN THE KIND OF
BRAIN-GUT ACCESS LITERATURE
WHICH IS REALLY NOW BRAIN-GUT
ACCESS IS A VERY HOT TOPIC.
IF YOU THINK ABOUT THE
PERSPECTIVE OF DIAGNOSIS, A
LITTLE BIT MORE, YOU MAY RECALL
WILLIAM OSLER SAID JUST LISTEN
TO YOUR PATIENT, HE IS TELLING
YOU THE DIAGNOSIS.
AND, YES, WE LISTEN TO OUR
PATIENT, AND THERE'S A LOT TO BE
DISCERNED FROM THAT CLINICIAN
INTERVIEW, WITH THE PATIENT.
BUT WE CAN LISTEN TO OTHER
THINGS AS WELL NOW.
SO AROUND 2016 THERE WAS A
PUBLICATION IN JAMA, WHERE
GOOGLE USED ITS PLAIN A.I.
ALGORITHM TO DIAGNOSE DIABETIC
RETINOPATHY FROM RETINAL SCANS,
FROM PHOTOGRAPHS OF THE RETINA.
AND TO STAGE IT AS WELL.
AND IN A THEME THAT WE'VE SEEN
AGAIN AND AGAIN, IN THE MEDICAL
LITERATURE, THE A.I.
OUTPERFORMED THE
OPHTHALMOLOGISTS.
TO UPDATE OSLER , I WILL LISTEN
TOKING YOUR PATIENT, HE'S
TELLING YOU THE DIAGNOSIS, WE
MIGHT WANT TO THINK LISTEN TO
ONE MILLION ANNOTATED
PHOTOGRAPHIC SCANS.
THEY TELL YOU THE DIAGNOSIS.
AND WE HAVE THE CAPACITY BECAUSE
OF MANY THINGS SEAN WAS TALKING
ABOUT, WE HAVEY A.I., CLOUD
COMPUTING, COMPUTATION,
INEXPENSIVE STORAGE, NEW MEANS
OF MULTI-MODAL DATA CAPTURE SO
WE CAN DO THIS.
TURNS OUT THAT THE BACKGROUND OF
INFORMATION UPON WHICH A
DIAGNOSIS IS MADE IS CRITICALLY
IMPORTANT, OFTEN NOT
QUANTITATIVELY CAPTURED.
FOR EXAMPLE THERE WAS A STUDY BY
RAJ MONRAY AND TEAM IN THE NEW
ENGLAND JOURNAL THAT SHOWED
PATIENTS WITH HYPERTROPHIC
CARDIOMYOPATHY, WHICH HAD
GENOMIC VARIANTS PRESUMED TO BE
PATH IGG KNOW MONIC IF YOU HAD
THE VARIANT, THAT HAD TO DO THE
BACKGROUND, CONTEXT IN WHICH
DIAGNOSES WERE BEING MADE AND
CONTEXT TRUE WHICH DATASETS WERE
PUT TOGETHER.
THIS IDEA OF PRIOR PROBABILITY
IS NOT WELL HANDLED IN OUR
HEALTHCARE SYSTEM.
AND PAPER THAT I PUBLISHED IN AN
N
ALS, WHEN THE KID HAS A SORE
THROAT, THE QUESTION IS, IS
STREP GOING AROUND.
IF IT'S NOT, IT'S PROBABLY NOT
STREP.
IN THE HUGE DATASET WE TOOK A
LOOK TO SEE WHAT THE PROBABILITY
OF A TRUE POSITIVE STREP TEST
WAS, WHETHER STREP WAS GOING
AROUND OR STREP WAS NOT GOING
AROUND, IN THAT LOCAL AREA.
AND WE WERE ABLE TO ADJUST FOR
VARIABLE WE MADE UP, INCREDIBLY
SIMPLE, CALLED THE LOCAL
PROPORTION POSITIVE, WHICH IS
JUST POSITIVE OVER NEGATIVE OF
STREP AND WHEN WE ADJUSTED FOR
THAT WE GOT MUCH MORE ACCURATE
DIAGNOSTICS.
THIS IS SOMETHING EXTREMELY
SIMPLE TO DO, IT WAS A FRACTION,
THIRD GRADE MATH, NOT SOMETHING
WE'RE ABLE TO DO AT SCALE IN THE
HEALTH SYSTEM BECAUSE THE DATA
DON'T COME TO THE RIGHT PLACES.
I'LL GIVE AN EXAMPLE FROM DINNER
THE OTHER NIGHT.
A FAMILY, A CHILD WAS JUST BACK
FROM COSTA RICA, AND HAD SOME
DISCOMFORT IN THE CHEST.
CHEST X-RAY SHOWED NODULES.
THE DOCTOR, NOT A PEDIATRIC,
DOCTOR SAID METASTATIC SARCOMA
FROM A PRIMARY THAT WE HAVEN'T
YET IDENTIFIED, CAN GO THROUGH
THREE WEEKS OF TESTING, DID NOT
DO THE PRIOR PROBABILITY THING,
TAKING INTO ACCOUNT THAT THE
CHILD WAS JUST BACK FROM COSTA
RICA, AND IT WAS A TROPICAL
INFECTION.
SO, THE BACKGROUND IS INCREDIBLY
IMPORTANT, AND WE'RE FLAWED AS
CLINICIANS, I SPEAK BEING NO
DIFFERENT FROM OTHER CLINICIANS
IN THIS RESPECT, I DON'T SEE
PATIENTS ANYMORE BUT I DID, WE
CAN'T DO IT ALL IN OUR HEAD.
SO, I WROTE A PIECE IN JAMA
THAT'S CALLED THE EVOLUTION OF
PATIENT DIAGNOSIS FROM ART TO
DIGITAL DATA-DRIVEN SCIENCE.
THE IDEA THERE WAS THAT WE NEED
TO USE DATA FROM LARGE NUMBERS
OF PATIENTS LIKE GOOGLE WITH THE
RETINAL PHOTOGRAPHS AS ONE
EXAMPLE TO TAKE CARE OF EVEN ONE
PATIENT.
A MILLION SCANS LETS US TAKE
CARE OF ONE PATIENT, MUCH MORE
ACCURATELY AND POTENTIALLY MUCH
MORE EFFICIENTLY.
IN ORDER TO DO THIS WE NEED A
COMPACT WITH OUR PATIENTS, I
BELIEVE, SO THEY UNDERSTAND THAT
THEIR DATA ARE BEING USED ON
THEIR BEHALF AND ON THE BEHALF
OF OTHER PATIENTS, AND THAT THEY
ONLY WANT TO BE TREATED IN
HEALTHCARE SYSTEMS WHERE DATA
ARE BEING USED THIS WAY.
I THINK IT WILL AT SOME POINT IN
THE NOT TOO DISTANT FUTURE
BECOME AN ABSOLUTE EXPECTATION
THAT DIAGNOSTIC PROCESSES WILL
USE LARGE SCALE DATA.
NOW, LET'S THINK FOR A MOMENT
WHY DOESN'T THAT HAPPEN NOW?
ARE KEN AND SEAN THE ONLY TWO
PEOPLE WHO THOUGHT OF THIS?
NO.
PEOPLE KNOW THAT THIS NEEDS TO
HAPPEN, BUT IT'S HARD.
IT'S HARD TO GET DATA TO COMBINE
ACROSS SITES OF CARE.
IT'S VERY HARD TO GET DATA BEING
IN THE RIGHT PLACE AT THE RIGHT
TIME.
AND IT'S VERY HARD TO GET
CLINICIANS WITHIN A HOSPITAL TO
SHARE DATA WITH EACH OTHER, IT'S
VERY HARD TO GET HOSPITALS AND
PRACTICES TO SHARE DATA MORE
WIDELY.
SO, LET ME GIVE YOU A COUPLE
EXAMPLES OF HOW I THINK THIS
BEGINS TO -- IS BEGINNING TO
UNFOLD, IN POSITIVE AND
FORWARD-LOOKING WAYS.
SO WHEN THE STIMULUS PACKAGE
AFTER THE 2008 RECESSION WAS
PASSED, ONE PIECE OF THAT WAS
SOMETHING CALLED THE HIGH TECH
ACT.
AND THE HIGH TECH ACT GAVE A LOT
OF MONEY TO A LITTLE KNOWN
GOVERNMENT AGENCY CALLED THE
OFFICE OF THE NATIONAL
COORDINATOR OF HEALTH
INFORMATION TECHNOLOGY, VERY
WELL KNOWN TO SOME OF US BUT
LESS WIDELY KNOWN TO SOME OTHER
GOVERNMENT AGENCIES, GAVE $48
BILLION TO THIS AGENCY TO
PROMOTE ADOPTION OF ELECTRONIC
MEDICAL RECORDS.
I WROTE A PIECE IN THE JOURNAL
AT THE TIME WITH ZAK COHINIK,
PROPOSED IF YOU'RE GOING TO
PROMOTE ALLS OF ELECTRONIC
MEDICAL RECORDS YOU SHOULD DO
WHAT STEVE JOBS HAD DONE WITH
THE iPHONE, JUST A YEAR
BEFORE, WHICH WAS TO CREATE AN
APP STORE.
HOW DID STEVE JOBS CREATE AN APP
STORE?
HE CREATED AN APP STORE BY USING
SOMETHING CALLED AN APPLICATION
PROGRAMMING INTERFACE, API.
THE API MEANS THAT YOU CAN
CONNECT APPS TO THE iPHONE,
EVERYONE'S DONE THIS, YOU ALL
KNOW HOW TO DO THIS, BUT THE
REASON IT'S SO EASY TO DO IS
BECAUSE THERE'S A PROGRAMMING
INTERFACE, AN API, THAT MEANS
THE DEVELOPER OF THE APP, GOOGLE
MAPS OR SOMETHING ELSE, DO THE
NOT NEED TO MEET WITH STEVE
JOBS.
THEY USE THE API, THE APP CAN
ASK FOR CONTACTS FROM THE PHONE,
CAN ASK FOR OTHER THINGS, FROM
THE PHONE, THAT MIGHT BE USEFUL,
FOR THAT APP TO WORK.
GPS COORDINATES, LATITUDE AND
LONGITUDE, ACCELEROMETER, THINGS
LIKE THAT.
SO THE API LETS THE APP GET DATA
FROM THE PHONE.
WE PROPOSE THERE SHOULD BE AN
API IN HEALTHCARE RECORDS SO AN
INNOVATOR COULD GET TO THE POINT
OF CARE SO THAT AN INNOVATOR
COULD CREATE AN APP, CONNECT IT
TO AN ELECTRONIC HEALTH RECORD,
VERY EASILY.
ALSO SO A CLINICIAN USING AN APP
COULD HAVE A DIFFERENT INTERFACE
FOR THE ELECTRONIC MEDICAL
RECORDS SO AN OBSTETRICIAN AND
PEDIATRICS COULD HAVE TWO VIEWS,
NEEDING A DIFFERENT SET OF
INFORMATION.
OBSTETRICIAN HAS A MOTHER TO BE,
AND AN EMBRYO TO TAKE CARE OF,
AND DATA ON BOTH.
AND PEDIATRICIAN HAS A DIFFERENT
SET OF CRITERIA, AND A
DERMATOLOGIST ANOTHER SET,
NEUROLOGIST ANOTHER SET.
THE IDEA HERE IS THAT WITH APPS
THAT CONNECT TO THE ELECTRONIC
HEALTH RECORD THROUGH AN API YOU
CAN FIRST OF ALL HAVE RAPID
DEPLOYMENT OF INNOVATIONS,
SECOND OF ALL YOU CAN HAVE
DIFFERENT EXPERIENCES FOR
DIFFERENT CLINICIANS, AND THAT'S
FOR ONE PATIENT AT A TIME.
AND IN ONE PATIENT AT A TIME API
WE CREATED SMART ON FIRE, LONG
STORY, BUT WE ACTUALLY WERE
FUNDED TO DO THIS.
WE DID IT.
WE EVEN GOT IT INTO SOME OF A
REGULATORY REGIME THROUGH THE
21ST CENTURY CURES ACT.
SMART ON FIRE, THIS API, LETS
YOU CONNECT APPS TO THE HEALTH
RECORD IS AIMED -- IS IN THE
21ST CENTURY CURES ACT AND THE
RULE SUPPORTING IT, CALLED CURES
ACT RULE.
ANY ELECTRONIC HEALTH RECORD IN
THE COUNTRY YOU CAN CONNECT AN
APP TO A PATIENT, CAN CONNECT AN
APP TO.
WHO DID THIS FIRST?
IRONICALLY WE MENTIONED THEM IN
OUR NEW ENGLAND JOURNAL PIECE
BUT IRONICALLY APPLE DID THIS
FIRST.
THEY WERE NOT ONLY THERE WITH
THEIR OWN API BUT WERE THE FIRST
USERS OF OUR API, AND THEY TOOK
THE HEALTH APP IN THE APPLE --
ON THE APPLE PHONE, THE WHITE
HEART WITH THE -- WHITE SQUARE
WITH RED HEART AND CONNECTED
HEALTH APP TO ALL ELECTRONIC
HEALTH RECORDS, SO THAT ANY
PATIENT CAN DOWNLOAD THEIR DATA
ONTO THEIR PHONE AND SHARE IT.
AND AMAZINGLY, THIS PHONE, THIS
DOWNLOAD, INCLUDES THE NOTES, IT
INCLUDES THE UNSTRUCTURED DATA.
AND AT THIS MOMENT IN TIME, FOR
PATIENTS TO BE ABLE TO GET FULL
ACCESS TO THEIR ELECTRONIC
HEALTH RECORD INFORMATION,
INCLUDING THE CLINICIAN NOTES,
ACROSS AN API FROM ANY
ELECTRONIC MEDICAL RECORD AND BY
THE WAY ALSO FROM ALL THE
DIFFERENT SITES OF CARE WHERE
THEY MAY GO, THEIR COMMUNITY
HOSPITAL, TERTIARY CARE
HOSPITAL, PRIMARY PRACTICE,
THEIR SPECIALIST PRACTICE, THEY
CAN GET THESE DATA FROM ALL OF
THOSE SITES AND ACTUALLY CREATE
A UNIFIED MEDICAL RECORD FOR
THEMSELVES, SHARE IT WITH THEIR
CLINICIANS, SHARE IT WITH
RESEARCHERS, SHARE WITH NIH
STUDIES.
THEY CAN SHARE IT WITH
CARETAKERS.
BUT POTENTIALLY MOST IMPORTANTLY
TODAY THEY CAN SHARE IT WITH
A.I.
AND IF THEY CAN SHARE IT WITH
RESPONSIBLE A.I., OR THEY CAN
JUST TAKE RESPONSIBILITY ON
THEMSELVES AND THEY CAN SHARE IT
WITH ChatGPT ON THEIR PHONE,
AND ALL OF A SUDDEN THEY'VE GOT
AN A.I. COMPANION WHO HAS ACCESS
TO THEIR FULL ELECTRONIC HEALTH
RECORD INFORMATION, THIS IS PART
OF THE BEGINNING OF A VERY
INTERESTING DIRECT-TO-CONSUMER
HEALTHCARE ECOSYSTEM THAT'S
EMERGING.
LET ME TELL YOU ABOUT ANOTHER
ASPECT OF THIS API.
WE HAVE A SECOND API, WHICH LETS
YOU GET POPULATION DATA OUT OF
THE ELECTRONIC HEALTH RECORD.
AND THE API THAT LET'S YOU GET
HEALTH -- POPULATION LEVEL OUT
OF A HEALTH RECORD ALSO IN THE
SAME FORMAT, FORMAT CALLED FHIR,
IT'S A STANDARD DATA FORMAT,
THAT LETS THIRD PARTIES AND
THIRD PARTIES AND WE'VE
DEMONSTRATED THIS, INCLUDE, FOR
EXAMPLE, CENTERS FOR DISEASE
CONTROL, WE'RE TALKING TO THE
FDA, AND WE'RE PART OF SOMETHING
CALLED THE ARPA-H BIOMEDICAL
DATA FABRIC, WHERE WE'RE
CONNECTING HEALTH CARE SYSTEM TO
RESEARCH ENTERPRISE WITH THE CDC
WE WERE CONNECTING HEALTHCARE
ECOSYSTEM TO PUBLIC HEALTH
ENTERPRISE, AND WITH THE FDA TO
SURVEILLANCE, POST-MARKET
SURVEILLANCE ENTERPRISE.
BUT WITH THE VERY SAME DATA
COMING OUT IN A STANDARDIZED WAY
ANYWHERE IN THE HEALTHCARE
SYSTEM AND THIS IS FOR THE CIO
TO BE ABLE TO EASILY GET THESE
DATA OUT.
IF YOU'VE EVER DRIED TO GET DATA
OUT, YOU MIGHT KNOW IT'S
COMPLICATED AND NOT NECESSARILY
SHAREABLE IN THE SAME FORMAT IN
TWO DIFFERENT SITES.
THIS IS A STANDARD FORMAT, ONE
ANALYTIC PROCESS CAN RUN
ANYWHERE IN THE HEALTHCARE
SYSTEM.
SO VERY INTERESTING THINGS
HAPPENING.
THIS IS A PARTICULARLY OPPORTUNE
MOMENT TO DISCUSS THESE
TECHNOLOGIES.
I WAS UNEXPECTED AT A HIGH LEVEL
WITH A BUNCH OF NERDS AROUND A
TABLE AT HHS HEADQUARTERS LAST
WEEK, THEY INVITED PEOPLE TO
DISCUSS ISSUES RELATED TO A CMS
AND ALSO THIS NATIONAL
COORDINATOR OF HEALTH
INFORMATION TECHNOLOGY REQUEST
FOR INFORMATION ABOUT THESE VERY
APIs, SMART ON FHIR, AND CLEAR
TO ME THE ADMINISTRATION IS VERY
MUCH PLANNING TO USE THESE
APIs TO MOVE HEALTH DATA
AROUND THE SYSTEM, INCLUDING
DIRECTLY TO PATIENTS, TO EMPOWER
PATIENTS WITH THEIR OWN
INFORMATION.
AND IN SOME WAYS, THERE IS ALSO,
AS FOLKS MAY KNOW, THIS
ADMINISTRATION HAS A
DEREGULATORY STANCE, AND I AND
OTHERS HAVE CALLED FOR IF THERE
IS GOING TO BE DEREGULATION IN
THE HEALTHCARE SPACE, WE THINK
THAT REGULATING INSTEAD OF EHR
VARIOUS EHR FUNCTIONALITIES,
JUST KEEPING WITH THESE
REGULATIONS, MAKING SURE DATA
CAN GO IN AND OUT OF ELECTRONIC
MEDICAL RECORDS, STANDARDIZED
FORMATS, REGULAR
INTEROPERABILITY INSTEAD OF USER
INTERFACE AND THINGS ON A
ELECTRONIC HEALTH RECORD, MIGHT
BE A MOMENT WHERE WE SEE
ACTUALLY A DEREGULATORY ACTION
THAT IMPROVES THE USE OF
ELECTRONIC HEALTH RECORD DATA
FOR MANY OF THE NICHD
OBJECTIVES.
IT'S TURNED OUT THAT'S -- THAT
THIS KIND OF INTERFACE WHERE YOU
CAN GET DATA OUT, MOFFITT
AROUND, SHARE IT, THAT'S ONE
PART OF INTEROPERABILITY.
ANOTHER PART IS SOMETHING I'VE
BEEN EXPLORING AND WITH SOME
SUCCESS IN A NETWORK OF
CHILDREN'S HOSPITALS WE HAVE SIX
AND SOON SEVEN CHILDREN'S
HOSPITALS, WHO ARE USING
INTEROPERABLE DATA, WHERE WE
HAVE -- IN A FEDERATED NETWORK.
AND THIS COMPARED TO A
CENTRALIZED NETWORK WHERE ALL
THE DATA ARE STORED IN ONE
DATABASE, IN A FEDERATED NETWORK
EACH SITE INSTALLED A LITTLE BIT
OF SOFTWARE AND KEEPS ALL THEIR
DATA ELECTRONIC HEALTH RECORD
DATA, CONSENTED GENOMIC DATA,
OTHER TYPES OF OMICS DATA, SOON
EXPOSOME DATA, ALL LOCALLY, ALL
HAPPENS LOCALLY.
A.I. PROCESSING NOTES,
EXTRACTING ADDITIONAL
INFORMATION ALL LOCALLY BUT WE
CAN QUERY ACROSS ALL THOSE SITES
AND SEE WHERE INTERESTING
PATIENTS ARE AND BRING DOWN
PARTICULAR COHORTS OF DATA ON
CONSENTED PATIENTS, FOR EXAMPLE,
AND ALSO WITH ELECTRONIC HEALTH
RECORD DATA, THROUGH WAIVERS OF
CONSENT, LARGE POPULATION DATA
IN STANDARDIZED FORMAT WE CAN
SHARE FOR PURPOSES OF RESEARCH.
AND WE'VE EXPLORED OTHER TYPES
OF INTEROPERABILITY IN THIS
NETWORK SO WE HAVE COMMON
APPROACHES TO CONSENT, 145,000
CONSENTED PATIENT SO FAR, COMMON
APPROACH TO ELECTRONIC HEALTH
RECORD DATA STORAGE ACROSS 16
MILLION PATIENTS' RECORDS,
COMMON APPROACHES TO
REPRESENTING GENOMIC DATA ACROSS
ABOUT, SO FAR, 40,000, SOON TO
BE MORE LIKE 100,000 GENOMES.
MY DEPARTMENT CHAIR ASKED ME
RECENTLY CAN WE PARTICIPATE AS A
SITE IN A GENE THERAPY TRIAL FOR
PITT-HOPKINS SYNDROME, A TCF 4
GENE MUTATION, A RARE NEUROLOGIC
DISORDER.
USING QUERIES WE WERE ABLE TO
IDENTIFY SCORES OF PATIENTS WITH
THIS EXCEPTIONALLY RARE DISEASE
WHO NOW HAVE THE BENEFIT OF
BEING OFFERED CLINICAL TRIALS.
SO WE SEE THE BENEFITS OF THIS
KIND OF APPROACH EMERGING VERY
QUICKLY AND THAT'S JUST ONE
EXAMPLE OF THE TYPE OF WORK THAT
WE'RE ABLE TO DO IN A NETWORK.
I'LL GIVE ONE MORE EXAMPLE AND
I'LL PAUSE IN CASE THERE'S
DISCUSSION THAT WE MIGHT HAVE.
BUT ANOTHER EXAMPLE IS LOOKING
ACROSS THIS WHOLE NETWORK FOR
PATIENTS WHO ARE ON DIAGNOSTIC
ODYSSEYS, THAT ARE POTENTIALLY
DETECTABLE THROUGH CERTAIN TYPES
OF SIGNATURES IN THEIR
TRAJECTORIES, OR THEIR OMICS
DATA, OR CLUSTERS OF SYMPTOMS OR
LABORATORY FINDINGS.
WE'VE BEEN LOOKING PARTICULARLY
IN THIS SPACE OF RARE PULMONARY
DISEASE, FOR EXAMPLE UNDIAGNOSED
BRONCHIECCASIS, INTERSTITIAL
LUNG DISEASE, AND I'LL GIVE THE
LAST EXAMPLE WE'RE BEGINNING TO
WORK ON, HOW DO WE TAKE ALL
THOSE DATA THAT WE HAVE AND,
AGAIN, GO BACK TO THE
RELATIONSHIP BETWEEN POPULATION
DATA AND INDIVIDUALS, HOW DO WE
REFLECT THOSE DATA INTO
SOMETHING LIKE A CASE
CONFERENCE?
SORT OF LIKE A TUMOR BOARD BUT
COULD BE IN A PULMONARY DISEASE
CLINIC OR INFLAMMATORY BOWEL
DISEASE CLINIC, HOW DO WE
REFLECT LARGE AMOUNTS OF DATA
AND WITH THE USE OF OTHER TYPES
OF A.I. TO SUPPORT CASE
CONFERENCE, WITH A BUNCH OF US
SITTING AROUND A TABLE TRYING TO
HELP EACH OTHER WITH OUR
KNOWLEDGE AND KNOWLEDGE OF THE
LITERATURE AND EXPERIENCE WITH
PATIENTS, TO SORT OF DIRECT THE
NEXT SET OF THERAPEUTIC OPTIONS
FOR PATIENT OR SORT THROUGH
DIAGNOSIS, HOW DO WE DO THAT IN
A FULLY INFORMATICALLY SUPPORTED
WAY.
SO THESE ARE SOME THINGS ON THE
SORT OF CUTTING EDGE OF THE
THINGS THAT WE'RE TRYING TO DO.
BUT IT'S A VERY INTERESTING
MOMENT IN REGULATED
INTEROPERABILITY.
I'M GLAD THIS KIND OF -- THIS
SYNERGY WITH THE POLICY WORLD ON
THIS RIGHT NOW AND CURRENT HHS
MAKES IT A VERY GOOD TIME TO BE
ABLE TO HAVE THIS DISCUSSION AND
THIS OPEN SESSION OF THE NICHD
COUNCIL.
THANK YOU VERY MUCH.
AGAIN, THANK YOU FOR INVITING M.
>> THAT WAS TREMENDOUS.
I WILL SAY ALSO THE RIFFING NOT
ONLY FROM DR. MOONEY'S
PRESENTATION BUT ALSO THE
COUNCIL'S AWARE WE'VE WORKED A
LOT WITH THE OFFICE OF THE
NATIONAL COORDINATOR THROUGH THE
HHS PATIENT-CENTERED OUTCOMES
RESEARCH TRUST FUND, INCLUDING
THE LING FHIR, MOMS AND BABIES,
THE GUIDE, AND VALERIE COTTON
AND REBEKAH ROSEN WORKING ON
FHIR RESOURCES FOR SOME OF OUR
DATA ASSETS.
I THINK THIS IS SOMEWHERE WE'RE
MOVING AND HAVE BEEN MOVING FOR
QUITE SOME TIME AND WE MAY
ACTUALLY WANT TO FOLLOW UP WITH
YOU ON SOME DISCUSSIONS YOU'RE
HAVING.
WE MEET REGULARLY WITH CMS AND
ONC.
IT WOULD BE GOOD TO KNOW WHAT'S
BEING DISCUSSED SO WE CAN
ANTICIPATE THOSE CHANGES.
I WILL ALLOW ONE QUESTION.
WE'D LIKE TO GIVE A TEN-MINUTE
BREAK, I DON'T WANT TO TOTALLY
CLOSE DOWN DISCUSSION BUT WANT
TO KEEP US SLIGHTLY ON SCHEDULE.
DOES ANYONE ON COUNCIL HAVE A
QUESTION FOR DR. MANDL?
SO, BONNIE, DR. MALDONADO?
>> YEAH, THANK YOU SO MUCH.
THIS IS REALLY EXCITING
PRESENTATION.
CAN YOU TALK JUST A BIT ABOUT
SECURITY, GIVEN THAT WE WORRY
ABOUT UPLOADING DATA THAT CAN BE
SHARED BROADLY AND ACCESSED FROM
OUTSIDE PARTIES TO PERSONALIZED
DATA, I'D LIKE TO HEAR A LITTLE
BIT MORE ABOUT THAT.
>> SURE.
IT'S A GREAT QUESTION.
A VERY COMMON QUESTION I GET
AFTER MY PRESENTATIONS.
SO, I'LL ANSWER IT SUCCINCTLY,
OF COURSE THERE'S MYRIAD ISSUES,
BUT LET ME ASSURE YOU THAT WHAT
WE'RE DOING PUTS PRIVACY AND
PATIENT CONTROL FIRST, IS NOT AN
AFTERTHOUGHT AND CERTAINLY NOT
SOMETHING THAT'S BEEN IGNORED.
IN THE ONE PATIENT AT A TIME,
FOR EXAMPLE, THE APPLE HEALTH
APP, ENABLING YOU TO DOWNLOAD
YOUR DATA TO YOUR PHONE, IF YOU
TAKE APPLE AS AN EXAMPLE, THEY
HAVE GONE PRIVACY FIRST TO AN
EXTREME DEGREE, IN A LAUDABLE
WAY.
DATA YOU DOWNLOAD TO YOUR PHONE
IS ONLY ON YOUR PERSONAL DEVICE,
WHICH HOPEFULLY YOU HAVE A
PASSWORD ON, PASSWORD
PROTECTION, AND IN YOUR PERSONAL
I-CLOUD.
APPLE CANNOT SEE IT.
NO ONE CAN SEE IT, UNTIL YOU
SHARE IT.
ONCE YOU -- IF YOU DO SHARE IT
WITH A THIRD PARTY, NOW JUST
LIKE EVERYTHING ELSE YOU SHARE
WITH A THIRD PARTY, THERE ARE
TERMS OF SERVICE THAT MAY OR MAY
NOT BE BENEFICIAL, THAT BECOMES
A FEDERAL TRADE COMMISSION
QUESTION.
IN THE POPULATION DATA SHARING,
THOSE DATA ARE ONLY BEING
ACCESSED ACROSS THIS API BY THE
SAME CHIEF INFORMATION OFFICERS,
ET CETERA, IN THE I.T.
DEPARTMENTS.
THEY ARE JUST ABLE TO DO IT MUCH
MORE EASILY, GET THE DATA IN A
STANDARDIZED WAY, AND THEN SHARE
IT SUBSEQUENTLY ACCORDING TO
APPROPRIATE DATA SHARING
POLICIES, IF IT'S RESEARCH, IRB,
IF IT'S PUBLIC HEALTH AUTHORITY,
PUBLIC HEALTH AUTHORITY, SO THE
TECHNOLOGY ITSELF DOES NOT
PRESENT PRIVACY ISSUES.
BUT THE WAY THAT IT'S USED JUST
LIKE THE WAY ANY TECHNOLOGY IS
USED, MIGHT PRESENT PRIVACY
ISSUES.
I'LL MENTION ONE MORE THING IN
THE TECHNOLOGY THAT WE'RE USING
IN ALL THESE PROJECTS I
MENTIONED FOR POPULATION HEALTH,
WE USE A VERY NOVEL PRIVACY
PRESERVING APPROACH WHERE THE
DATA ARE ONLY AGGREGATE COUNTS
AND YET CONTAIN HUGE AMOUNT OF
INFORMATION.
AND THIS HAS BEEN SOMETHING THAT
HAS BEEN VERY SUCCESSFUL
EXPERIMENT.
I'LL HOLD IT RIGHT THERE.
>> THANK YOU SO MUCH.
WITH THAT I WANT TO SAY THIS IS
SOMETHING WE'VE BEEN THINKING
ABOUT QUITE A LOT HERE AND WE'LL
CONTINUE THESE DISCUSSIONS
RELATED TO DATA AND CONTINUE TO
TRACK.
THANK YOU AGAIN, DR. MANDL, FOR
JOINING US.
THANK YOU FOR THE DISCUSSION.
WE'LL COME BACK, LET'S GIVE IT
2:16, PLEASE TAKE A MINUTE TO
TAKE AN ACTUAL BREAK AND WE WILL
BE BACK TO START UP AGAIN WITH DR. ESPINOSA.
THANK YOU VERY MUCH.
>> I WAS EVEN MORE GENEROUS,
2:17.
IS DR. ESPINOSA WITH US?
GREAT.
THANK YOU.
WELCOME BACK.
THANK YOU FOR STICKING WITH US
THROUGH A VERY SHORT BREAK.
IT IS NOW A PLEASURE TO
INTRODUCE DR. JOAQUIN ESPINOSA
ON TRISOMY 21, A NEW CLINICAL
TRIALS TESTING SAFETY AND
EFFICACY OF IMMUNOMODULATORY
THERAPIES TO TREAT DIVERSE
CO-OCCURRING CONDITIONS IN THIS
POPULATION.
EXECUTIVE DIRECTOR AT THE
INSTITUTE FOR DOWN SYNDROME,
UNIVERSITY OF COLORADO,
PROFESSOR IN DEPARTMENT OF
PHARMACOLOGY, UNIVERSITY OF
COLORADO SCHOOL OF MEDICINE,
HOLDS ANNA AND JOHN ENDOWED
CHAIR, RECEIVED A Ph.D. FROM
UNIVERSITY OF BUENOS AIRES,
ARGENTINA, IMMUNE DYSREGULATION
IN DOWN SYNDROME, MECHANISMS IN
CLINICAL TRIALS.
WELCOME, GREAT TO SEE YOU AGAIN.
THANK YOU FOR JOINING US.
>> THANK YOU VERY MUCH.
HOW IS THE AUDIO THIS AFTERNOON?
>> PERFECT, SIR.
THANK YOU.
>> GREAT.
HOW ABOUT THE FIRST SLIDE, IS
THAT VISIBLE?
PERFECT.
YOU'RE READY TO PROCEED.
>> LET ME SET UP A TIMER TO MAKE
SURE I DON'T OVEREXTEND MY
WELCOME.
THANK YOU VERY MUCH FOR THE
INVITATION AND OPPORTUNITY TO
SHARE WITH YOU SOME OF THE
RESEARCH FUNDED BY NIH HERE AT
THE LINDA CRNIC INSTITUTE FOR
DOWN SYNDROME.
I WOULD LIKE TO START BY
CELEBRATING PEOPLE WITH DOWN
SYNDROMEA DIVERSE POPULATION
SURPASSING ALL EXPECTATIONS,
MORE PERSONS ALIVE WITH DOWN
SYNDROME THAN EVER BEFORE,
LIVING LONGER THAN EVER BEFORE.
INDIVIDUALS WITH DOWN SYNDROME
ARE DEFINED BY OUTDATED NOTIONS
ABOUT WHAT IS POSSIBLE WHEN
LIVING WITH AN EXTRA CHROMOSOME.
WHO WOULD HAVE THOUGHT PEOPLE
WOULD BE PROTAGONISTS IN A TV
SHOW THAT WON MULTIPLE EMMY
AWARDS, MAIN CHARACTERS IN
HOLLYWOOD MOVIES, AUTHORS,
MUSICIANS, PUBLIC SPEAKERS, SO
ON.
THEY ARE CHALLENGING US TO THINK
DIFFERENTLY ABOUT THIS
CONDITION.
I WOULD LIKE TO EMPHASIZE DOWN
SYNDROME IS NOT A RARE DISEASE.
IT'S NEITHER RARE NOR A DISEASE.
IT'S A MEDICAL CONDITION THAT'S
THE LEADING CAUSE OF
INTELLECTUAL DEVELOPMENT
DISABILITY IN THE HUMAN
POPULATION, THIS GRAPH NEEDS
SOME UPDATING, IS ILLUSTRATING,
IF YOU LOOK AT THE AGE BRACKET
TO YOUNG TO OLD THE BIRTH RATE
HAS NOT DECREASED, NOT IN THE
USA, BECAUSE OF LARGE GAINS IN
LIFE EXPECTANCY SIGNIFICANT
GROWTH OF THE POPULATION WITH
DOWN SYNDROME, WHO HAVE NEW
PHENOMENON IN THE UNIVERSE,
ADULTS WITH DOWN SYNDROME.
NOW IT IS NO LONGER A RARITY.
A NEWBORN WITH DOWN SYNDROME
TODAY IN THE US IS EXPECTED TO
LIVE UP TO 60 YEARS OR SO, SO
CLEARLY HERE TO STAY.
NOW, THE CAUSE OF DOWN SYNDROME
HAS BEEN KNOWN SINCE THE LATE
'50s, AN EXTRA COPY OF
CHROMOSOME 21, SEQUENCED IN
2000, NOT MUTATIONS IN THE
STRICT SENSE, JUST AN EXTRA
COPY, 50% MORE OF THESE 225
GENES.
THE OBVIOUS QUESTION FOR US AS
SCIENTISTS HOW DOES THIS CAUSE
THE HALLMARKS OF DOWN SYNDROME?
NEW UNDERSTANDING OF DOWN
SYNDROME HAS BECOME CLEAR THAT
BEYOND THE TRAITS, DEVELOPMENTAL
DEDELAYS, EARLY AGING, PEOPLE
WITH DOWN SYNDROME HAVE A
DIFFERENT CLINICAL RISK PROFILE,
LESS LIKELY TO DEVELOP RARE
CONDITIONS, FOR EXAMPLE BREAST
CANCER, PROSTATE CANCER,
PROTECTED FROM HYPERTENSION,
HIGHLY DISPOSEsED TO
ALZHEIMER'S AND OTHER
CONDITIONS, YOU WANT TO HELP
PEOPLE WITH DOWN SYNDROME LIVE
LONGER HEALTHIER LIVES, WE MUST
STUDY CO-OCCURRING CONDITIONS OF
DOWN SYNDROME.
WITHIN THE FRAMEWORK HAD A
INFLECTION POINT IN 2017,
CONGRESSIONAL HEARING, ON DOWN
SYNDROME, WE EXPLAINED
OBSERVATIONS, A PRODUCTIVE
COLLABORATION WITH CONGRESS, THE
FOUNDATION AND OFFICERS LEADING
TO CRE ACE OF THE "INCLUDE"
PROJECT.
THE STAR OF THE FRANK STEPHENS,
I'M A MAN WITH DOWN SYNDROME,
AND MY LIFE IS WORTH LIVING.
PLEASE STUDY MY EXTRA
CHROMOSOME, A GIFT TO SCIENCE, A
GIFT TO HUMANKIND.
SO FOLLOWING YEAR IN 2018, NIH
LAUNCHED THE INCLUDE PROJECT, A
TRANS-NIH PROJECT TO INCREASE
RESEARCH ON DOWN SYNDROME, THIS
CHART SHOWS BEFORE "INCLUDE,"
INVESTMENT WAS $20 TO $30
MILLION, NOW WITH "INCLUDE" $130
MILLION.
21 INSTITUTES PARTICIPATING, 400
AWARDS, HUNDREDS OF
PUBLICATIONS.
BASIC SCIENCE, COHORT
DEVELOPMENT, CLINICAL TRIALS.
I WOULD LIKE TO SHOW THE POWER
OF THE INCLUDE PROJECT AND THREE
COMPONENT STRUCTURE, FROM THE
DISCOVERY TO CLINICAL TRIALS,
DISCOVERY WAS ORIGINALLY
REPORTED IN 2016 LEADING THEN
WITH "INCLUDE" PROJECT TO TRIAL
IN 2020 SHOWING ACTIVITIES WHICH
LED TO A SECOND CLINICAL TRIAL
UNDERWAY, NOW A THIRD AND FOURTH
UNDER PREPARATION, SUPPORTED BY
MULTIPLE NIH INSTITUTES FOR
"INCLUDE."
COMPONENT 2, COHORT STUDIES, WE
HAVE A COHORT STUDY, THE HUMAN
TRIAL PROJECT, 1400 PARTICIPANTS
SINCE 2016.
WE SUPPORT "INCLUDE" PROJECT
GENERATING THIS OMICS DATASETS,
THEN SHARED WITH INCLUDE DATA,
INCLUDING PROJECT ADHERES TO
OPEN SCIENCE PRINCIPLES, DATA IS
SHARED.
PROJECTS HAVE IN TURN SECURED
NIH FUNDING LEADING TO MANY
PUBLICATIONS.
ONE KEY OBSERVATION FROM THIS
COHORT STUDY NOW REPRODUCED IN
OTHER COHORT STUDIES, THERE'S
WIDESPREAD AUTOIMMUNITY IN
PEOPLE WITH DOWN SYNDROME.
75% OF ADULTS HAVE BEEN
DIAGNOSED WITH AN AUTOIMMUNE
DECISION, MOST COMMON IS THYROID
DISEASE.
SOMETHING MORE RECENTLY
APPRECIATED IS HIGH PREVALENCE
OF ALOPECIA, ATOPIC DERMATITIS,
PSORIASIS.
TEN TIMES MORE COMMON IN DOWN
SYNDROME RELATIVE TO THE GENERAL
POPULATION, CELIAC DISEASE.
I SHOULD POINT OUT RELEVANT TO
NICHD THAT 80% OF THESE
CONDITIONS ARE DIAGNOSED DURING
PEDIATRIC AGE.
SO THE TITLE OF THE PAPER SAYS
TRISOMY 21 ACTIVATES INTERFERON
RESPONSE, SIGNALING IS PART OF
THE INNATE IMMUNE SYSTEM TO
FIGHT OUR VIRUSES, INTERFERONS
HAVE SITE -- CYTOKINES, TRIGGER
EGGER IMMUNE CELLS.
TOO MUCH OF THIS GOOD THING
COULD BE BAD, SPECIFICALLY
LEADING TO RISK OF AUTOIMMUNITY.
WHY WOULD PEOPLE WITH DOWN
SYNDROME HAVE HYPERACTIVE
INTERFERON SIGNALING IN FOUR
INTERFERON RECEPTOR GENES ON
CHROMOSOME 21, THREE TYPES OF
INTERFERON SIGNAL, DIFFERENT
LIGANDS AND RECEPTORS, TRIGGER
JAK/STAT CASCADE, LEADING TO
IMMUNE RESPONSE.
THE HYPOTHESIS HERE IS THIS
RESPONSE INCREASES JAK/STAT
SIGNALING OVEREXPRESSION CHRONIC
IMMUNE ACTIVATION AND
AUTOINFLAMMATION COULD IN TURN
CONTRIBUTE TO CLINICAL
DEVELOPMENT HALLMARKS OF DOWN
SYNDROME.
LET ME SHOW ONE PIECE OF DATA
FROM THIS PAPER TO PUT IN
PERSPECTIVE.
THIS GRAPH SHOWS INTERFERON
COURSE, MEASURING THOSE
INTERFERON STIMULATED GENES,
GIVING RESEARCH PARTICIPANTS A
VALUE, PARTICIPANTS IN THE
GENERAL POPULATION, THE
CONTROLS, LOOK AT PARTICIPANTS
WITH DOWN SYNDROME, MORE THAN
TEN-FOLD HIGHER INTERFERON
SCORES WITH STRONG VIABILITY FOR
COMPARISON PURPOSES I'M SHOWING
HERE THE SAME INTERFERON SCORES
IN THE GENERAL POPULATION
WITHOUT AND WITH COVID-19.
LOOK AT THE SCALES.
THE SAME SCALE.
IN OTHER WORDS, BY THIS METRIC
THE BLOOD OF PEOPLE WITH DOWN
SYNDROME LOOKS LIKE THE BLOOD OF
A PERSON HOSPITALIZED WITH
COVID-19.
SO, FOLLOW-UP PUBLICATION,
PLASMA PROTEOMICS ANALYSIS, MANY
INFLAMMATORY FACTORS WE KNOW ARE
DRIVERS OF AUTOIMMUNE
CONDITIONS, BY THIS METRIC THE
BLOOD OF INDIVIDUALS WITH
TRISOMY 21 RESEMBLES BLOOD OF
THOSE IN THE GENERAL POPULATION
AFFECTED BY AUTO-INFLAMMATORY
CONDITIONS, FOR EXAMPLE, SAY
RHEUMATOID ARTHRITIS.
SUPER CHARGED WITH SUPPORT FROM
THE "INCLUDE" PROJECT WE
EMBARKED MORE AMBITIOUS EFFORTS
ALWAYS IN THE HUMAN TRIALS
PROJECT, REPORTING THE MAP OF
THE IMMUNE SYSTEM SHOWING GLOBAL
IMMUNE REMODEL, HYPERSENSITIVITY
TO INTERFERONS, IT'S HARD TO
FIND AN ASPECT OF THE IMMUNE
SYSTEM THAT'S NOT DIFFERENT IN
DOWN SYNDROME.
MANY IMMUNE CELL TYPES ARE
INCREASING FREQUENCY,
PRO-INFLAMMATORY, AUTOIMMUNITY,
THAT COMES AT EXPENSE, MANY
OTHER CELLS BECOME EXHAUSTED,
OVER A LIFETIME.
ALL ADULTS WITH DOWN SYNDROME
ARE IMMUNOCOMPROMISED, SENSITIVE
TO LUNG INFECTIONS.
WE WERE ABLE TO DEMONSTRATE
PROFOUND METABOLIC
DYSREGULATION.
THIS IS DONE THROUGH PLASMA
METABOLOMICS ANALYSIS INCLUDING
WHAT WE CALL IMMUNOMETABOLOMIC
PATHWAYS, THIS CONVERTS
TRYPTOPHAN INTO NEUROTOXIC
CATABOLITES, IF THEY GET TO YOUR
BRAIN CAUSE DAMAGE, SEIZURES,
EXCITATORY TOXICITY.
THIS COULD PROVIDE A LINK
BETWEEN INFLAMMATION AND
NEUROLOGICAL PHENOTYPES IN DOWN
SYNDROME.
THERE ARE MANY MORE
PUBLICATIONS, BY US AND OTHERS,
THIS IS NOW AN ENTIRE FIELD OF
RESEARCH.
THE POINT I'M MAKING IS
INTERFERON SIGNALING IS
HYPERACTIVE IN DOWN SYNDROME.
THE QUESTION IS SO WHAT?
WHAT ARE THE IMPACTS?
HYPERACTIVE INTERFERON IN DOWN
SYNDROME, COULD IT BE UNDERSTOOD
IN PART AS AN INTERFERONOPATHY?
WE MOVE TO COMPONENT ONE, BASIC
SCIENCE APPROACHES.
EARLY WORK FROM THE BIANCHI LAB
AT NIH DOCUMENTED IN MOUSE
MODELS CARRYING THREE COPIES OF
RECEPTORS.
WE ASKED WHAT WOULD HAPPEN IF WE
COULD NORMALIZE COPY NUMBERS
JUST FOR RECEPTORS, AND THE
TITLE SAYS IT ALL.
TRIPLICATION OF LOCUS
CONTRIBUTES TO HALLMARKS OF DOWN
SYNDROME IN MOUSE MODEL.
SO I'D LIKE TO SHOW ONE PIECE OF
DATA BECAUSE THIS IS ONE OF THE
AMAZING THINGS WE CAN LEARN IN
THE 21ST CENTURY IN THE LAB.
CRISPR GENOME EDITING TECHNOLOGY
WE DELETED ONE COPY OF THIS
INTERFERON GENE CLUSTER, THOSE
INTERFERON RECEPTORS THAT WERE
OVEREXPRESSED IN THE MOUSE MODEL
MOUSE MODEL NOW THEY ARE NO
LONGER OVEREXPRESSED IN THAT
ANIMAL THAT'S BEEN ENGINEERED.
OTHER TRIPLICATED GENES LIKE
THOSE SHOWN HERE REMAIN
OVEREXPRESSED.
ALLOWS YOU CONTINUE OF RECEPTORS
RELATIVE TO GENES THAT ARE
TRIPLICATED.
RECEPTORS CONTINUE TO
DYSREGULATION ACROSS EVERY
TISSUE, CONFER CONTRIBUTE TO
HYPERSENSITIVITY, CRANIAL
ABNORMALITIES, INTERFERONOPATHY
STARTS IN THE WOMB BECAUSE
CONGENITAL FORMATION, FACIAL
ABNORMALITIES ARE EMBRYONIC
SIGNAL TYPES.
WE GOT VERY LUCKY HERE, THE
PHARMACEUTICAL INDUSTRY HAS
DEVELOPED JAK INHIBITORS, SOME
SHOWN HERE, NOT A COMPREHENSIVE
TABLE.
MANY BRAND NAMES, TARGET JAK
KINASES.
THE PLUS SYMBOL INDICATES
APPROVAL FOR THAT CONDITION.
AS YOU CAN SEE MEDICINESES ARE
USED BY PHYSICIANS IN MANY
DISCIPLINES.
COULD THIS JAK INHIBITOR
NORMALIZE IN DOWN SYNDROME?
SO WE WOULD NOT BE AFFECTING THE
LIGANDS, MESSING WITH RECEPTORS,
SMALL MOLECULES BLOCKING
PATHWAYS, NEED TO BE TAKEN
DAILY, SHORT HALF-LIFE IN THE
BODY, CLEARED RAPIDLY IF ONE
NEEDS TO REVERSE THE
INTERVENTION.
NOW COMPONENT THERE, CLINICAL
TRIALS, TREATING FIVE IMMUNE
CONDITIONS IN A SINGLE TRIAL,
ALL THESE CONDITIONS SHOWN HERE,
MORE COMMON IN DOWN SYNDROME,
35% OF ADULTS WITH DOWN SYNDROME
AFFECTED BY AT LEAST ONE,
PROTOCOL FOUR TO NINE MONTHS OF
TREATMENT WITH FDA-APPROVED
INHIBITOR THAT IS NOT APPROVED,
APPROVED FOR ARTHRITIS.
>> IF YOU CAN HEAR ME YOU FROZE
ON OUR SIDE.
WE'RE JUST WAITING FOR YOU TO
CATCH BACK UP WITH US.
WE CAN HEAR YOU.
WE NEED TO RESHARE YOUR SLIDES.
>> VERY GOOD.
>> OR WE CAN PULL THEM UP IF YOU
SAY NEXT SLIDE.
LET ME KNOW WHICH WAY.
>> GO AHEAD.
SHARE AGAIN.
>> OKAY.
THERE WAS A BIT OF AN
INTERACTION THERE.
>> YES, OKAY.
THAT'S WHY WE LIKE TECHNOLOGY,
BECAUSE IT'S SEAMLESS.
>> HERE WE GO.
>> THERE IT IS.
NOW WE'RE GOOD.
PERFECT.
>> VERY GOOD.
SO WE'RE SHARING ABOUT THE
CLINICAL TRIALS PROTOCOL,
ENDPOINTS, SKIN PATHOLOGY, AND
CONDITION AND QUALITY OF LIFE
WHILE WE'RE AT IT.
OPEN LABEL, EVERYONE RECEIVED
THE MEDICINE.
GOAL WAS 40 PARTICIPANTS, WITH
ONE JAK INHIBITOR.
STRONG REPRESENTATION OF
MINORITIES, 25% HISPANIC, 15%
BLACK OR BIRACIAL.
WE WERE ABLE TO BRING MANY
FAMILIES FROM ACROSS THE COUNTRY
TO COLORADO TO PARTICIPATE IN
THE TRIAL.
MOST COMMON QUALIFYING
CONDITIONS WERE HIDRANETIS,
ALOPECIA, PSORIASIS.
THE MEDICINE IS SAFE TO EMPLOY
IN DOWN SYNDROME.
THIS SHOWS THE MOST COMMON
ADVERSE EVENTSES, AFFECTING ONLY
A MINORITY, RUNNY NOSE, COUGH,
WEIGHT GAIN.
WEIGHT CHANGES, SKIN RASH, A
SINGLE ADVERSE EVENT, 27.7 YEARS
OFFING ONS VASE, AN EPISODE OF
THROMBOEMBOLISM WITH
CONTRACEPTIVE PILLS, PARTICIPANT
RECOVERED FAVORABLY.
TEN-FOLD INCREASE INTERFERON
ACTIVITY, PARTICIPANTS IN
CLINICAL TRIAL START AT
BASELINE, AVERAGE FOR DOWN
SYNDROME, AND THEN THEY COME
DOWN TO WITHIN THE NORMAL RANGE,
EIGHT TO NINE-FOLD.
IMMUNOMODULATING, NOT
NECESSARILY SUPPRESSING THE
IMMUNE SYSTEM.
JAK INHIBITOR REDUCED CYTOKINE
SCORES, IMMUNE METABOLITES IN
THE PATHWAY.
HOW ABOUT THE SKIN?
LOOK AT THIS.
A YOUNG MAN FROM TEXAS COMING TO
COLORADO.
86% OF SCALP IS AFFECTED AT
BASELINE, YOU SEE NOW HE CAN
GROW THE BEAUTIFUL RED HAIR.
WE KNOW WHY HIS FRIENDS CALL HIM
ED SHEERAN.
ANOTHER PARTICIPANT, CLEAR WHEN
GOING ON THIS MEDICINE.
THIS PARTICIPANT WAS SO
INCAPACITATED COULDN'T COME TO
COLORADO, WAS GIVEN MEDICINE BY
A COLLEAGUE IN VERMONT.
YOU WILL HEAR LATER TODAY ABOUT
THIS.
WHAT ABOUT CONDITION?
WE MEASURED A LOT OF THINGS.
WORKING MEMORY, EPISODIC AND
SPECIAL MEMORY, MOTOR CONTROL,
DEVELOPMENT, QUALITY OF LIFE.
WITHOUT A PLACEBO CONTROL, WE
CANNOT DISCARD PLACEBO EFFECTS,
TREATMENT EFFECTS BUT
SOMETIMES -- LOOK AT THIS LADY,
28 YEARS OLD, REGRESSION
DISORDER, THIS IS BASELINE.
WE'RE ASKING HER TO DRAW THE
PATH OF THE CAR AND MOTORCYCLE
WITH PENCIL, SHE'S NOT FULLY
COMPREHENDING.
SHE'S COLORING.
AT 16 WEEKS SHE WAS IN A MUCH
BETTER SPACE.
BEFORE TREATMENT RECEIVING
ELECTRO CONVULSIVE TREMENDOUS,
AND AT THE END A MEDICINE WAS
PRESCRIBED, WE'RE TRYING TO
REPURPOSE FOR SKIN PURPOSES IN
DOWN SYNDROME, THIS WOMAN NOW
TAKING IT FOR REGRESSION
DISORDER
WHAT IS THAT?
IT'S A CONDITION CHARACTERIZED
BY RAPID ONSET, DAYS, WEEKS,
CATATONIA, HALLUCINATIONS, A
SUBSET OF THESE CASES SCIENCE
CASES ARE ASSOCIATED WITH IMMUNE
DYSREGULATION AFFECTING THE
BRAIN SIMILAR TO WHAT WE KNOW IN
THE GENERAL POPULATION,
ENCEPHALITIS, THESE ARE SOME OF
THE PAPERS PROVIDING EVIDENCE
FOR THE HYPOTHESIS.
IT WAS FAMILIES AND MOMS THAT
REALLY ASKED QUESTIONS AND MADE
CONNECTIONS BRINGING TO THE
PROFESSIONALS SUCH AS DR.
SANTORA LEADING TO THE FIRST
RANDOMIZED CLINICAL TRIAL FOR
REGRESSION DISORDER, STUDY
COVERED ON THE COVER OF THE
WASHINGTON POST, THEY DID A GOOD
JOB AT RECOGNIZING THE MOMS AND
YOU'LL HEAR FROM LINDA IN A FEW
MINUTES.
NOW WE'RE IN THE BUSINESS OF
THERAPIES FOR REGRESSION, IMMUNE
THERAPIES VERSUS INTERVENOUS
IMMUNOGLOBULIN, LORAZEPAM, WITH
LOS ANGELES AND COLORADO, KEY
MEMBERS.
REPURPOSE DRUGS APPROVED FOR
OTHER CONDITIONS, ALL BENEFITED
SOME PEOPLE WITH DOWN SYNDROME,
SO WE'RE RANDOMIZING
PARTICIPANTS TO THE THREE ARMS,
THEY DO KNOW WHAT THEY ARE
GETTING.
THERE'S A DELAYED TREATMENT
GROUP.
THE WHOLE POINT HERE IS TO
ARRIVE TO A PERSONALIZED
MEDICINE APPROACH, WHO WOULD
BENEFIT THE MOST FROM WHICH
MEDICINE?
WE KNOW OF PARTICIPANTS THAT
BENEFITED FROM EACH AND THAT
HAVEN'T BENEFITED FROM EACH.
WHAT ARE THE CHARACTERISTICS
THAT PREDICT A GOOD RESPONSE,
BIOMARKERS TO HELP PARTICIPANTS,
SPECIFIC CONDITIONS.
IN CONCLUSION, INTERFERON
HYPERACTIVITY CAUSES ISSUES,
AUTOIMMUNE DISORDERS OBVIOUSLY,
ALSO RECOMPLICATION FROM LUNG
INFECTION, AND MORE TANTALIZING
CONTRIBUTING TO NEUROLOGIC
ASPECTS, LEADING TO FIRST INKIND
CLINICAL TRIALS, RESTORING
IMMUNE BALANCE COULD HAVE
BENEFITS IN THIS POPULATION.
MANY QUESTIONS, LONG-TERM
SAFETY, WHAT ARE POSSIBLE
BENEFITS, HOW MUCH MAY HAVE AN
IMMUNE BASIS?
HOW EARLY SHOULD WE START THIS
TREATMENT?
IS TREATMENT EVEN POSSIBLE?
DR. BIANCHI WAS VERY
ENTHUSIASTIC ABOUT THE CONCEPT.
PRENATAL TREATMENT.
SHOULD EVERYONE WITH DOWN
SYNDROME BE TREATED OR WITH
OBVIOUS AUTOIMMUNE DIAGNOSIS, A
LOT OF RESEARCH AHEAD.
I PRESENTED WORK FROM A HUNDRED
INDIVIDUALS, IF I WROTE THEM ALL
DOWN YOU WOULDN'T BE ABLE TO
READ IT.
I'LL TAKE A MOMENT TO
ACKNOWLEDGE THE PROGRAM
LEADERSHIP, ALL RESEARCH
PARTICIPANTS AND FAMILIES, MANY
WONDERFUL COLLABORATORS, THE
"INCLUDE" PROJECT AND
PHILANTHROPIC PARTNERS.
I HOPE THERE'S TIMES FOR CREST
QUESTIONS AND CRITICISMS.
>> THAT WAS OUTSTANDING.
THANK YOU SO MUCH.
IT'S A TRANSFORMATIVE PROGRAM
AND I THINK JUST REALLY
DEMONSTRATES THE KEY
CONTRIBUTIONS OF EACH OF THE
COMPONENTS OF THE "INCLUDE"
PROGRAM AND ALSO THE CLEAR
BENEFITS OF THE TEAM SCIENCE
YOU'VE USED.
LET ME OPEN TO QUESTIONS FROM
THE COUNCIL.
RAISE YOUR HAND SO I CAN SEE OR
COME OFF MUTE AND PUT YOUR VIDEO
ON IF YOU HAVE A QUESTION.
ARE YOU SEEING HANDS?
I'M NOT SEEING ANY.
HERE WE GO.
DR. JABS?
>> YEAH, THAT WAS MARVELOUS.
THAT'S VERY EXCITING.
CAN I ASK YOU, DID YOU SEE AN
AGE DEPENDENT EFFECT WITH YOUR
CLINICAL TRIAL?
>> WE DID NOT.
INTERFERON HYPERACTIVITY OCCURS
ACROSS THE LIFESPAN.
IT STARTS AS EARLY AS WE CAN
MEASURE IT, IN YOUNG INFANTS.
THERE IS AN ASPECT OF
INFLAMMATION OF DOWN SYNDROME
THAT'S AGE DEPENDENT, I WOULD
SAY MINOR ASPECT.
WE ALL DEVELOP SO-CALLED INFLAME
AGING AS WE AGE.
WHAT WE DO OBSERVE IS THAT THE
BENEFIT OF THE MEDICINE, THIS IS
PROBABLY TRUE FOR BOTH THE SKIN
CONDITIONS AND REGRESSION, THE
EFFECT IS GREATER WHEN THE
TREATMENT OCCURS EARLY, RIGHT?
IF MANY YEARS HAVE GONE BY SINCE
THE ONSET OF SYMPTOMS, THE
MEDICINE MAY HAVE A HARDER TIME
PROVIDING BENEFIT.
BUT AS OF THIS POINT WE DON'T
HAVE ANY OBVIOUS AGE-DEPENDENT
EFFECTS.
>> THANK YOU.
>> OTHER QUESTIONS?
OKAY.
I WILL ACKNOWLEDGE THIS IS A
MAJOR ADVANCE, I REALLY DO
APPRECIATE THE FACT THAT HAVING
WATCHED THIS TALK NOW IN TWO
FORMS A LITTLE BIT TWICE, I WILL
SAY JUST THE LEVEL OF
INTEGRATION THAT YOU ALL HAVE
HAD AND ABILITY TO CONDUCT
TRIALS HAS BEEN TRANSFORMATIVE
AND I LOOK FORWARD TO SEEING
WHAT YOU AND YOUR COLLEAGUES
WILL BE DOING MOVING FORWARD.
I KNOW THAT DR. BHATTACHARYA HAS
BECOME FAMILIAR WITH THIS
PROJECT AND SO HE HAS RECEIVED A
BRIEFING AND HAS SEEN ADVANCES,
ALSO FELT IT WAS REALLY
IMPACTFUL.
THANK YOU FOR ALL YOU'RE DOING.
>> THANK YOU.
>> SO, NOW I WANT TO THANK DR.
ESPINOSA, ALSO MOVE NOW BECAUSE
FOR THOSE FAMILIAR WITH OUR
COUNCIL, YOU KNOW THAT WE
REGULARLY FEATURE A SEGMENT
CALLED VOICE OF THE PARTICIPANT
WHICH GIVES A CHANCE TO HEAR
FROM THOSE WHO HAD PERSONAL
EXPERIENCES RELATED TO RESEARCH
NICHD FUNDS, AND TODAY WE
WELCOME MISS ANGELA LOMBARDO AND
MISS LINDA ROAN.
ANGELA IS A PARENT OF ADULT SON
WITH DOWN SYNDROME AND
AUTOIMMUNE CONDITION WHO
PARTICIPATED IN CLINICAL TRIALS.
LINDA ROAN IS COMMUNITY LIAISON,
BUT HERE AS A PARENT OF ADULT
DAUGHTER WITH AUTOIMMUNE SKIN
CONDITION AND DOWN SYNDROME
REGRESSION DISORDER WHO
BENEFITED FROM HER PARTICIPATION
IN RESEARCH TRIALS.
I WANT TO SAY THANK YOU FOR
JOINING US TODAY.
WE'LL START WITH THE LOMBARDO
FAMILY.
ANGELA, IF YOU WOULD START FOR
US WE WOULD APPRECIATE IT.
THANK YOU FOR JOINING TODAY.
>> OF COURSE.
THANK YOU FOR HAVING US.
I WANT TO TELL YOU ABOUT ISAIAH,
AND ABOUT OUR EXPERIENCES AND
HISTORY WITH SKIN ISSUES AND OUR
PARTICIPATION IN THE CLINICAL
TRIALS WITH DR. ESPINOSA AND
TEAM.
HISTORY, 29 YEARS OLD, BORN 2
1/2 MONTHS PREMATURE.
HE HAD HIRSCHBERG DISEASE,
PERMANENT COLOSTOMY BAG, A SMALL
HOLE IN THE HEART WENT AWAY BY
AGE 3.
MOST OF HIS LIFE WAS COLOSTOMY
AND G.I. ISSUES.
IN HIGH SCHOOL THINGS CHANGED.
WE NOTICED HIS SKIN STARTED TO
CHANGE A LOT IN HIGH SCHOOL.
HE HAD REALLY BAD ECZEMA.
HE HAD PATCHES OF HAIR THAT
STARTED TO BE GONE, QUARTER-SIZE
PATCHES THAT STARTED TO GO AWAY
AND STARTED GETTING THESE BOILS
BUT THEY WERE ABSCESSES THAT HE
WOULD GET OFTEN.
ISAIAH HAS BEEN EMPLOYED SINCE A
JUNIOR IN HIGH SCHOOL, WAS VERY
BUSY IN HIS LIFE.
HE'S DANCED, HE WAS IN HORSEBACK
RIDING, HE WAS DOING BASKETBALL.
I KEPT HIM VERY ACTIVE IN HIS
COMMUNITY.
BUT IN HIGH SCHOOL HE STOPPED
HORSEBACK RIDING BECAUSE OF
MULTIPLE SURGERIES TO HIS BUM
FOR THE CYSTS THAT WOULD DEVELOP
ON HIS BUM.
HE WOULD MISS TIME FROM SCHOOL
BECAUSE HE COULDN'T SIT IN A
CHAIR WITHOUT PAIN.
HE WOULD MISS TIME FROM SCHOOL
BECAUSE IT HURT TO MOVE HIS ARM.
ISAIAH NEVER REALLY COMPLAINED
ABOUT WHAT WAS HAPPENING BECAUSE
HE BECAME AFRAID OF THE
TREATMENTS OF THE PROBLEM.
SO HE'S HAD A GAZILLION -- THEY
EXCISED WOUNDS, WOULD CUT HIS
SKIN OPEN TO DRAIN IT, PACK IT,
OVER AND OVER AND AND THEN IT
WOULD GO AWAY AND DOM BACK.
SO, HE LEARNED TO LIKE PROTECT
HIMSELF, WHICH WAS GOOD THAT HE
LEARNED TO PROTECT HIMSELF BUT I
DIDN'T KNOW THERE WAS PROBLEMS.
HE WOULD WALK LIKE THIS AND I
COULD SEE HE WOULDN'T USE HIS
ARM.
WHEN YOU LIFTED THE ARM IT WAS
LIKE A TANGERINE-SIZE ABSCESS,
SEVEN SURGERIES TO ONE ARMPIT
AND A FEW TO THE OTHER IN
ADDITION TO EXCISIONS.
THEY WENT IN WHEN HE WAS IN HIS
EARLY 20s AND DID A FULL
CLEANOUT, TOOK OUT SWEAT GLANDS
AND WOULD STILL GET ABSCESSES.
ISAIAH STOPPED RECREATION
BECAUSE OF THAT, ECZEMA AND
PSORIASIS WOULD IMPACT HIM, HE
ALWAYS HAD FLAKES ON , ITCHING,
COULDN'T WORK IN THE FOOD
INDUSTRY.
PEOPLE WOULD SAY YOU HAVE
DANDRUFF.
HE DIDN'T.
HIS SKIN WOULD FLAKE AND FALL
OFF A LOT.
WE TRIED, I SPENT SO MUCH MONEY
ON LOTIONS, CREAMS, OINTMENTS,
GOT AN ADULT PROVIDER FOR
DERMATOLOGY, THEY GAVE HUMERA
SHOTS THAT NEVER DID ANYTHING.
WE WERE AT A LOSS, WOULD KEEP
BUYING OINTMENTS AND CREAMS THAT
NEVER IMPROVED HIS SKIN.
I WORK IN A HOSPITAL IN A DOWN
SYNDROME PROGRAM.
WE GO TO NATIONAL CONFERENCES
EVERY YEAR SO I WAS IN A TALK
FOR PROFESSIONALS AT THE
CONFERENCE, AND DR. ESPINOSA
SHARED HIS STUDY.
I GOT SIX TEXT MESSAGES FROM
PEOPLE GOING, GET ISAIAH TO HIM,
TALK TO THAT MAN ABOUT ISAIAH!
EVERYONE KNEW HIS HISTORY WITH
SKIN ISSUES.
AND I REALLY DIDN'T APPROACH HIM
BECAUSE I WAS, YOU KNOW, VERY
AWARE OF THE BOUNDARY OF
PROFESSIONAL AND PERSONAL USE OF
WHAT I WAS ACCESSING
INFORMATION, BUT WE WENT TO THE
BOOTH AT THE CONFERENCE, AND
TALKED TO THEM, AND I WAS, TO BE
HONEST, HESITANT, I'M A SINGLE
MOM.
I WORK, ISAIAH WORKS, BUT WE'RE
NOT ROLLING IN IT LIKE ISAIAH
WOULD SAY, I COULD NOT AFFORD
COLORADO, COULD NOT IMAGINE
TAKING TIME OFF WORK FOR THIS.
BUT WE LEARNED ABOUT IT AND THE
TRAVEL WAS COVERED WHICH WAS
HUGE.
AND I STILL WAS HESITANT JUST
BECAUSE IT SEEMED A LOT TO DO
WITH SUCH BUSY LIVES.
BUT I THINK WE JUST DECIDED LIKE
WE HAD TO GO FAR BECAUSE WHAT WE
WERE DOING WASN'T WORKING AND WE
WERE DESPERATE TO GIVE HIM
PEACE, TO HOLD YOUR SON DOWN IN
THE E.R. WHEN SOMEONE CUTS HIM
WITHOUT SEDATION AND THEY SAY
IT'S A LITTLE KNICK, BUT IT'S
NOT GOOD, NOT HEALTHY FOR HIM TO
BE TRAUMATIZED LIKE THAT SO
MUCH.
SO WE WENT FOR IT.
AND WE STARTED GOING TO COLORADO
A LOT.
IT WAS AMAZING.
I HAVE BEEN ON ONE SIDE OF
RESEARCH, NEVER AS A
PARTICIPANT, BUT WE WERE EXCITED
TO SEE IF ISAIAH WOULD BENEFIT,
THRILLED TO THINK THAT US
PARTICIPATING COULD HELP OTHER
PEOPLE.
SO IF WE COULD MAKE A DENT IN
WHAT THEY WERE LEARNING ABOUT
AND IF WE COULD HELP THEM GET
INFORMATION TO HELP FUTURE
FAMILIES AND FUTURE KIDS TO
PREVENT WHAT ISAIAH WENT THROUGH
AND HELP THEM, IT WOULD BE WORTH
IT FOR US.
WE TRAVELED TO COLORADO.
I WAS SO IMPRESSED WITH THE WAY
THEY SPOKE TO SAY ISAIAH,
CONSENTING PROCESS, MATERIALS TO
WORK WITH PEOPLE WITH
INTELLECTUAL DISABILITIES WAS SO
IMPRESSIVE.
SO ISAIAH TOOK THE MEDICINE AT
THE TRIAL, WE SAW IMPROVEMENTS
IMMEDIATELY.
LIKE IT WAS CRAZY.
I WAS ALWAYS TAKING PICTURES OF
HIS NECK, IT WAS MIND BLOWING
HOW MUCH WE SAW IMPROVEMENTS ON
HIS SKIN.
HE'LL STILL GET AN ABSCESS HERE
AND THERE BUT CONTROLLED WITH
HEAT, OPENS ON ITS OWN BUT
NOTHING.
HE HASN'T BEEN CUT SINCE WE
STARTED THE TRIAL, NO INCISIONS,
NO NEED BECAUSE IT'S BEEN
CONTROLLED.
THE TEAM WAS HELPFUL AND
OFFERING TO HELP US GET THE
MEDICINE COVERED.
WE WERE LUCKY HIS MEDICARE
COVERED IT FOR US.
BUT WE LEARNED QUICKLY THE
CHALLENGES WHEN THEY CHANGED OUR
MEDICARE PHARMACY PLAN, IT
WASN'T COVERED.
SO HE WENT THREE MONTHS WITHOUT
IT, THEY WANTED $1,800
CO-PAYMENT FOR EACH MONTH FOR
THE MEDICINE, I COULDN'T AFFORD
THAT SO HE CAME OFF OF IT.
AND WITHIN DAYS HIS SKIN GOT
PLAQUEY AGAIN, ABSCESSES IN THE
GROIN AREA, ARMPIT.
SO, IT WAS FRUSTRATING BUT WE
CHANGED HIS PHARMACY PLAN,
WAITED FOR THE TIME PERIOD, AND
WE WERE FINALLY ABLE TO GET BACK
ON IT.
HE'S ON IT.
IT'S JUST BEEN -- IT'S NIGHT AND
DAY.
WE'RE SO GRATEFUL.
IT'S CHANGED HIS LIFE.
I'M THANKFUL TO THE NIH,
"INCLUDE," THANKFUL TO THE
RESEARCHERS AND DR. ESPINOSA AND
TEAM.
I'M THANKFUL I WENT FOR IT.
SOMETIMES IT'S -- I REALLY
HESITANT BUT WE WENT FOR IT, AND
THE BEST DECISION I MADE FOR
ISAIAH IN A LONG TIME BECAUSE
IT'S REALLY IMPACTED HOW HE
LIVES AND IMPACTS HIS WORK.
AT HIS JOB HE WORKS FULL TIME,
FIVE DAYS A WEEK AT SHAKE SHACK,
A RESTAURANT.
THEY WEAR BLACK T-SHIRTS.
HIS JOB HAD TO GET A LINT ROLER
TO RUB ON HIM TO TAKE THE FLAKES
OFF SOMETIMES.
IT'S JUST NOT THE WAY IT SHOULD
BE.
I'M SO GRATEFUL THAT WE HAVE A
HANDLE ON IT AND WE WERE
PROVIDED THE OPPORTUNITY TO
ATTEND.
MY SON BENEFITED BUT I'M
THANKFUL WE WERE PROVIDED THE
OPPORTUNITY TO HELP WITH
RESEARCH, TO HELP OTHER FAMILIES
AND TO CONTRIBUTE TO THE
RESEARCH.
SO I THINK ISAIAH CHANGED,
CONFIDENCE, SELF-ESTEEM, IT
IMPACTS YOUR IDENTITY WHEN YOU
SEE YOURSELF AS DIFFERENT AND
ARE TREATED DIFFERENTLY BECAUSE
OF APPEARANCES, HOW HE LOOKED,
SKIN BEING ITCHY ALL THE TIME.
I THINK I'M FOREVER GRATEFUL AND
I THINK IT'S SO IMPORTANT TO
LOOK AT THE RESEARCH, ALSO
APPRECIATIVE OF KNOWING THE
HISTORY AND WHEN DR. ESPINOSA
SHOWED THE NUMBERS HOW MUCH WAS
FUNDED PRIOR TO THINK OF THE
BENEFITS FOR FUTURE BABIES AND
FOR KIDS BEING BORN NOW WHO HAVE
BETTER OUTCOMES AND
TRAJECTORIES, BETTER HEALTH
BECAUSE OF THE STUFF WE LEARN
TODAY.
I'LL STOP.
OH, ISAIAH WANTED TO SAY
SOMETHING REAL QUICK.
>> I HAD.
I WANTED TO SAY I'M VERY GLAD I
WAS ABLE TO JOIN THE STUDY IN
COLORADO.
I'M SO HAPPY THAT MY SKIN IS
HEALED.
I'VE GOTTEN BETTER.
IT WAS HARD HAVING SO MUCH PAIN
FROM SKIN ISSUES.
I LOVE TO WORK, BE WITH MY
FRIENDS.
MY SKIN CAUSED A LOT OF
TROUBLES.
I WAS EMBARRASSED ABOUT MY SKIN
SOMETIMES.
IT MADE ME SAD.
I'M THANKFUL WE WERE ABLE TO
PARTICIPATE, SO GLAD I'M TAKING
MEDICINE TO HELP MY SKIN AND
KEEP THE PIMPLES AWAY.
I FEEL PROUD ABLE TO HELP THIS
RESOURCE TO HELP OTHER PEOPLE
WITH DOWN SYNDROME AND NOT HAVE
THE PAIN WITH MY SKIN.
I LIKE WORKING WITH DR. ESPINOSA
AND TEAM.
THEY TALKED TO ME DIRECTLY,
EXPLAINED EVERYTHING PERFECTLY.
SO IT MAKES ME FEEL LIKE I'M
SEEN IN THE WORLD.
THANK YOU FOR LETTING ME SAY MY
STORY TODAY.
>> THANK YOU.
>> THANK YOU ALL SO MUCH FOR
SHARING YOUR STORY.
AND PARTICULARLY ABSOLUTE --
PARTICULARLY -- ISAIAH, I WANT
TO THANK YOU FOR SHARING YOUR
STORY IN SUCH A GREAT WAY.
>> THANK YOU.
>> WHAT IS THE THING THAT YOU
WENT BACK TO FIRST WHEN YOU FELT
BETTER, WHAT WAS THE THING THAT
YOU WANTED TO DO MORE THAN
ANYTHING?
>> I WANTED TO GO OUT WITH MY
FRIENDS MORE.
AND I LIKE TO MAKE MORE
CONNECTIONS WITH MY FRIENDS
WITHOUT BEING EMBARRASSED ABOUT
MY SKIN.
>> THAT SOUNDS LIKE THAT WAS A
HUGE IMPACT FOR YOU, SOMETHING
REALLY MEANINGFUL TO YOU.
AWESOME.
>> THANK YOU.
>> FANTASTIC.
ONE MORE QUESTION, WHEN YOU SAID
THAT THE TEAM TALKED TO YOU
INSTEAD OF YOUR MOM, EXPLAINED
EVERYTHING TO YOU, WERE THERE
THINGS THAT YOU WERE WORRIED
ABOUT OR SCARED OF THAT THEY
HELPED TO REASSURE YOU WERE
GOING TO BE OKAY?
>> YES.
I WAS SCARED ABOUT MY HAIR
BECAUSE WHEN I WAS YOUNGER, I
HAD BIG AFRO.
>> OH.
>> WHEN I WAS 7.
I WANTED IT BACK.
>> OH, THERE YOU GO.
>> BUT NOW IT'S IMPOSSIBLE TO
GET IT BACK.
THE PILLS, I GOT -- YOU HAVE
SIDE EFFECTS, BUT THAT WASN'T
THE CASE.
SO I'M PRETTY GLAD TO BE PART OF
THE TRIAL.
DR. ESPINOSA, I LOVE YOU AND YOU
ROCK!
>> FANTASTIC.
WE THINK HE ROCKS TOO.
WE'RE ON THE SAME PAGE.
>> THANK YOU.
>> I WANT TO MAKE SURE I CHECK,
WE HAVE ANOTHER 15 MINUTES, IF
THE OTHER FAMILY HAS JOINED US,
MISS LINDA ROAN HAS TIME.
ARE YOU ON, MS. ROAN?
I DON'T SEE HER ON TEAMS RIGHT NOW.
I'M SORRY ABOUT THAT.
I ENTERED THE MEETING THROUGH A
DIFFERENT ROUTE.
>> ALL GOOD.
>> A DIFFERENT LINK.
HERE I AM.
I'M SO SORRY ABOUT THAT.
MY APOLOGIES.
>> NO WORRIES.
>> IT IS SUCH A PLEASURE AND
HONOR TO BE HERE TODAY.
I CAN'T TELL YOU WHAT AN IMPACT
THE NATIONAL INSTITUTE OF
HEALTH, "INCLUDE" PROJECT, HAS
BEEN FOR MY DAUGHTER, BUT FOR MY
ENTIRE FAMILY.
WHAT I'D LIKE TO DO TODAY IS
TELL YOU MY DAUGHTER'S STORY
ABOUT DOWN SYNDROME REGRESSION
DISORDER.
I BELIEVE DANIEL HAS A LITTLE
FILM, IT'S VERY SHORT, ABOUT A
MINUTE, THAT SHOWS MY DAUGHTER
PRE-REGRESSION, DURING
REGRESSION, AND THEN
POST-REGRESSION.
AND IF THERE'S TIME THERE'S A
COUPLE VIGNETTES AND STORIES TO
SHARE FROM OTHER FAMILIES THAT
HAVE LOVED ONES WITH DOWN
SYNDROME REGRESSION DISORDER.
BUT LET ME GET STARTED WITH MY
FAMILY'S STORY.
MAYA IS 32 YEARS OLD NOW.
IN 2013, SHE WENT AWAY, WAS
ROCKING HER LIFE, HAVING A GREAT
LIFE.
AND SHE WENT AWAY TO A COLLEGE
PROGRAM FOR PEOPLE WITH
INTELLECTUAL DISABILITIES.
AND WHEN SHE CAME HOME FOR
THANKSGIVING, WE NOTICED SHE WAS
NOT HERSELF.
SHE WAS TALKING TO HERSELF UNDER
HER BREATH, HAD NO INTEREST IN
BEING WITH THE FAMILY, JUST NOT
HERSELF, TALKING LIKE SHE WAS IN
THIS TV SHOW THAT SHE LOVED.
CALLED PRETTY LITTLE LIARS, YOU
MAY REMEMBER IT FROM WAY BACK
WHEN.
WE CALLED THE SCHOOL.
THEY SAID THEY HAD NOT NOTICED
ANYTHING, SHE HAD BEEN DOING
GREAT.
WE DIDN'T SEND HER BACK A COUPLE
DAYS LATER.
THE SCHOOL CALLED AND SAID WE'VE
BEEN CHECKING AROUND, SHE'S BEEN
STAYING UP ALL NIGHT LONG,
COPYING FROM HER BOOK, SHE'S
BEEN COPYING OVER AND OVER.
SHE'S BEEN MISSING APPOINTMENTS.
NO, SHE'S NOT BEEN HERSELF.
WE TOOK HER TO THE CENTER FOR
DOWN SYNDROME.
THEY SAID WE DON'T KNOW WHAT
THIS IS.
IT DOESN'T HAVE A NAME.
JANUARY OF 2014.
WE KNOW IT'S A THING IN DOWN
SYNDROME.
WE STARTED WORKING WITH THE
PSYCHIATRIST HERE IN DENVER,
PSYCHOTROPIC MEDICINE.
I DON'T BELIEVE ANYTHING REALLY
WORKED BECAUSE IT WAS ABOUT 18
MONTHS BEFORE SHE STARTED
GETTING BETTER.
SHE WAS GOOD, REALLY GOOD, FOR
ABOUT SIX MONTHS.
AND THEN, WHAM, IN ONE DAY SIX
MONTHS AFTER SHE HAD GOTTEN MUCH
BETTER, IN ONE DAY SHE WAS BACK
TO NOT BEING ABLE TO EVEN SAY
HER NAME.
THIS IS A YOUNG WOMAN THAT WAS
VIBRANT, FULL OF LIFE, DOING
WELL.
WE TRIED ALL THE THINGS THAT WE
HAD DONE THE FIRST TIME BUT TO
NO AVAIL, NOTHING WORKED.
SHE WAS GETTING WORSE AND WORSE
AND WORSE.
SHE WAS -- I WAS HAND FEEDING
HER AT THIS POINT.
NOT TALKING AT ALL.
AND SO WE -- THE PSYCHIATRIST
SAID WE HAVE ONE MORE THING WE
CAN TRY.
WE THINK IT COULD BE ELECTRIC
CONVULSIVE THERAPY COULD HELP
HER.
NOW 2016, WE TRIED ECT, AND
WITHIN ONE MONTH SHE WAS BACK TO
HER OLD SELF, BACK TO HER
BASELINE SELF.
ALL WAS GOOD IN THE WORLD.
LIFE WENT ON.
SHE GOT A JOB.
TIME PASSES.
AND THEN IN 2020, BAM, AGAIN,
VERY, VERY ILL.
WE TRIED ALL THE THINGS AGAIN.
THIS TIME ECT DID NOT TOUCH IT.
SHE WAS BAD.
SHE WAS IN DEPENDS, I WAS HAND
FEEDING HER, I HAD A BUSINESS
WITH HER FATHER, WE HAD TO SHUT
THE BUSINESS DOWN BECAUSE IT WAS
A 24/7 ORDEAL TAKING CARE OF
HER.
AND I TALKED TO DR. SCOTTCO, HE
SAID THERE'S A DOCTOR IN
CALIFORNIA, DR. JONATHAN
SANTORO, LOOKING INTO THIS.
I SUGGEST YOU GO SEE HIM.
WE WENT TO CALIFORNIA, SAW DR.
SANTORO, AND HE SAID, YOU KNOW,
LINDA, HE RAN A LOT OF TESTS,
YOU SAID, YOU KNOW, I THINK WE
SHOULD TRY IVIG BUT YOUR
DAUGHTER HAS HAD THIS, WHATEVER
THIS IS, I THINK IT COULD BE AN
INFLAMMATORY PROCESS LIKE AN
AUTOIMMUNE ENCEPHALITIS.
WE DON'T KNOW ENOUGH TO SAY FOR
SURE BUT THAT'S WHAT MY THINKING
IS.
WE SHOULD GIVE IVIG A TRY, I'M
NOT SURE IT'S GOING TO WORK
BECAUSE SHE'S BEEN SICK FOR SO
LONG.
WE WENT BACK TO COLORADO, GOT
THE IVIG, SURE ENOUGH IT DID NOT
WORK.
BUT I REMEMBERED THE SCIENTIST
THAT I HAD TALKED TO IN THE
EARLY DAYS, THAT FIRST
REGRESSION, ASKING ME A BUNCH OF
QUESTIONS ABOUT MAYA'S IMMUNE
SYSTEM.
AND THEN I REMEMBERED.
AND DR. SANTORO WAS MENTIONING
HER IMMUNE SYSTEM.
AND SO I WENT, HMM, THESE TWO,
ONE SCIENTIST, ONE PHYSICIAN,
THEY NEED TO MEET.
I INTRODUCED THE TWO GENTLEMAN
AND THEY COMPARED NOTES AND AS I
THINK YOU ALL KNOW THEY HAVE
BEEN WORKING TOGETHER AND NOW
THERE'S A CLINICAL TRIAL LOOKING
AT DOWN SYNDROME REGRESSION
DISORDER.
AND SO DR. ESPINOSA SAID IS
THERE ANY CHANCE MAYA HAS AN
AUTOIMMUNE SKIN CONDITION?
NO, SHE'S GOT BUMPS THAT SHE
GETS IN HER GROIN AREA, THEY CAN
GET REALLY NASTY BUT HER PCP,
THEY WILL JUST LANCE THEM,
NOBODY MENTIONED IMMUNE SKIN
CONDITION.
HE SAID, HMM, I'D LIKE FOR YOU
TO SEE ONE OF THE DERMATOLOGISTS
WE WORK WITH.
SO WE DID.
SURE ENOUGH, IT WAS HS.
HE SAID LET'S GET MAYA IN THIS
TRIAL.
SHE QUALIFIES BECAUSE OF HER
SKIN CONDITION, BUT I HAD THIS
CURIOSITY, I'M WONDERING IF IT
MIGHT HELP HER BRAIN TOO.
SO OH MY GOSH, A GLIMMER OF
HOPE.
WE STARTED THE TRIAL, IT WAS A
16-WEEK TRIAL, SURE ENOUGH
MAYA'S BRAIN, HER SKIN WAS KIND
OF HELPED BUT HER BRAIN WAS
GREATLY HELPED.
AND SHE STARTED DOING WELL
AGAIN.
AND SO SHE NOW -- THAT WAS 2021.
AND NOW WE'RE IN 2025, THE
REASON SHE'S NOT HERE TODAY IS
BECAUSE SHE'S AT WORK.
SHE HAS TWO JOBS.
SHE HAS FRIENDS.
SHE JUST GOT BACK FROM
CALIFORNIA WITH SOME FRIENDS AND
HER HOST HOME PROVIDER LIVING --
JUST DOING GREAT.
SHE DOES OCCASIONALLY HAVE WHAT
WE CALL FLARES, BUT WITH THE
MEDICATION, TOPOCITINIB, ALSO
LORAZEPAM THAT WE'RE ALSO
STUDYING IN THE TRIAL, PERHAPS
AN ECT TREATMENT, SHE'S DOING
WELL, REALLY, REALLY WELL.
NOW WE'RE SEVERAL YEARS OUT.
I'M HOPEFUL THAT THIS WILL
CONTINUE AND WE HAVE EVERY
REASON TO BELIEVE THAT IT WILL.
I'D LIKE -- SEEING IS BETTER
THAN HEARING.
IT'S A MINUTE, A VIDEO THAT I'D
LIKE FOR YOU TO SEE NOW OF MAYA
PRE-REGRESSION, DURING
REGRESSION, AND MAYA
POST-REGRESSION.
SO, IF YOU COULD PLAY THAT
VIDEO, I THINK DANIEL HAS IT. THAT WOULD BE REALLY GREAT.
>> SHE HELPS ME UNDERSTAND MUCH
BETTER.
HI, IT'S ME, MIAH.
>> MIAH, DO YOU KNOW YOUR DAD'S
NAME?
CAN YOU TELL ME YOUR SISTERS'
NAMES?
WHAT HAVE YOU BEEN TALKING
ABOUT?
>> A LOT OF TIMES, I WANTED TO
LIKE DANCE MORE.
♪
I ALSO LIKE TO DESIGN CLOTHES.
I HAVE A JOB.
WAG'S CONSIGNMENT, IT'S LIKE A
FASHION EXTRAVAGANZA.
♪
>> I THINK YOU CAN SEE WHY WE'RE
SO, SO THANKFUL FOR THESE
CLINICAL TRIALS, AND THE PEOPLE
LIKE YOU THAT MAKE IT POSSIBLE.
I DON'T THINK I HAVE VERY MUCH
TIME LEFT BUT I'D LIKE TO LEAVE
YOU WITH ONE LITTLE STORY THAT'S
NOT ABOUT MY FAMILY.
IT'S ABOUT ANOTHER FAMILY,
SOMEONE I TALKED TO RECENTLY.
SO AT THE CRNICH INSTITUTE I WAS
CONTACTED BY A FAMILY A COUPLE
MONTHS AGO.
THEIR DAUGHTER, 15 YEARS OLD,
STARTED TO SHOW SIGNS OF
REGRESSION, THIS WAS IN
LOUISIANA.
THEY HAD NO IDEA WHAT REGRESSION
WAS.
OF COURSE, THEY FREAKED OUT.
WHAT'S GOING ON?
THEY TOOK HER TO THE HOSPITAL.
EMERGENCY ROOM.
AND THE EMERGENCY ROOM THREW
THEIR HANDS UP IN THE AIR, WE
DON'T KNOW WHAT THIS IS.
YOU KNOW, MAYBE IT'S DOWN
SYNDROME, WE DON'T KNOW.
SENT HER HOME.
A WEEK OR TWO WENT BY.
SHE WAS GETTING WORSE, WHICH
SOMETIMES WITH REGRESSION
DISORDER IT'S FAIRLY QUICK
ONSET, PEOPLE CAN DETERIORATE
QUICKLY.
SHE WAS GETTING WORSE AND WORSE
AND WORSE.
THEY WENT BACK TO THE EMERGENCY
ROOM, THIS TIME A DIFFERENT
HOSPITAL, A LARGER HOSPITAL.
AND THEY WENT BACK AND THEIR
DAUGHTER WAS, YOU KNOW, ADMITTED
INTO THE HOSPITAL.
THERE WAS A HOSPITALIST, SOME
DOCTOR WORKING IN THE HOSPITAL,
WHO SAID I DON'T KNOW WHAT THIS
IS.
BUT WENT INTO THE LITERATURE AND
STARTED SEEING ARTICLES FROM THE
RESEARCH ABOUT AUTOIMMUNITY IN
DOWN SYNDROME, ARTICLES FROM DR.
SANTORO ABOUT REGRESSION
DISORDER, OTHER PEOPLE IN THE
LITERATURE.
SO IN THE HOSPITAL, THIS YOUNG
WOMAN WHO HAD ONLY BEEN SICK FOR
A FEW WEEKS GOT IVIG, LEFT THE
HOSPITAL MUCH BETTER, HAS HAD
MORE IVIG, IS TOTALLY FINE.
WE'RE TALKING THIS JUST TOOK A
MATTER OF A FEW WEEKS OR A MONTH
OR SOMETHING, COUPLE MONTHS
VERSUS FOR SOME FAMILIES KIDS
HAVE BEEN SICK FOR DECADES, THEY
HAVE GIVEN UP.
THEIR KIDS LIVE IN BED.
SO IN A SHORT PERIOD OF TIME BIG
GAINS ARE BEING MADE AND
FAMILIES HAVE HOPE.
AND WE JUST CAN'T THANK YOU
ENOUGH.
THANK YOU.
>> WELL, FIRST OFF, THANK YOU
FOR COMING FIRST TO OUR COUNCIL
AND SHARING YOUR STORY AND YOUR
DAUGHTER'S STORY, AND THE STORY
OF OTHER FAMILIES.
ALSO THANK YOU FOR GETTING US
THE VIDEO.
IT'S TRANSFORMATIVE.
IF WE CAN, IF THE LOMBARDO
FAMILY AND MISS ROAN I'D LOVE TO
LET THE COUNCIL ASK ONE OR TWO
QUESTIONS, DO WE HAVE ANY
COUNCIL MEMBERS THAT WANT TO
ASK -- WE CAN DO TWO QUESTIONS,
I'M GOING TO ASK THE TEAM TO BE
FASTER.
THIS IS SO IMPACTFUL.
>> YES, THANK YOU SO MUCH TO
BOTH OF YOU FOR SHARING YOUR
STORY AND YOUR JOURNEY WITH
PARTICIPATING IN THE TRIAL.
IT'S VERY INSPIRING TO ALL OF US
TO HEAR FROM FAMILIES LIKE
YOURSELF.
WHAT ADVICE WOULD YOU GIVE TO
RESEARCHERS LIKE US BASED ON
YOUR EXPERIENCE TO ENROLL
FAMILIARS WITH DOWN SYNDROME OR
SIMILAR CONDITIONS IN RESEARCH?
>> I GUESS I'LL GO FIRST.
ANGELA, YOU CAN GO.
MY ADVICE TO RESEARCHERS WOULD
BE DON'T GIVE UP.
DON'T GIVE UP!
STAY WITH IT!
KEEP WORKING HARD!
YOU KNOW, I ALSO THINK THAT
PIECE OF ADVICE ABOUT SPEAKING
TO THE PERSON WITH THE
DISABILITY INCLUDING THEM IN THE
CONVERSATION IS HUGELY
IMPORTANT.
AND, YOU KNOW, PUBLISH, PUBLISH,
PUBLISH BECAUSE IT'S THOSE
ARTICLES THAT MAKE THE
DIFFERENCE.
>> YEAH, MY VOICE IS TERRIBLE.
I WAS GOING TO SAY THAT I THINK
IT'S DEFINITELY HOW DO YOU
ACCOMMODATE YOUR MATERIALS TO
SHARE AND HOW YOU TO TALK TO
PEOPLE AND GIVE THEM THE
MATERIALS AND INFORMATION.
IT'S IMPORTANT TO LIKE WE WOULD
HAVE NOT BEEN IN THE TRIAL
WITHOUT COSTS BEING INCLUDES,
HOW DO WE BREAK DOWN BARRIERS
ACCESS OR WE WOULD HAVE NEVER
BEEN ABLE TO DO IT.
>> THANK YOU.
ANY OTHER QUESTIONS FROM THE
COUNCIL?
WE CAN DO ONE MORE.
>> SO I WANT TO THANK ALL OF YOU
SO MUCH FOR JOINING US THIS
AFTERNOON.
PRESENTATIONS ARE HONESTLY WHAT
KEEP ME GOING FROM QUARTER TO
QUARTER.
AND REALLY DO MAKE SUCH AN
IMPACT ON OUR STAFF.
THIS HAS BEEN COMPLETELY MISSION
CONCORDANT, SOMETHING WE DO
EVERY DAY, ONE OF THE FOUNDING
THINGS THAT IS REALLY DEAR TO US
AT NICHD.
SO DR. ESPINOSA, YOUR CONTINUED
WORK AND PARTNERSHIP, ESPECIALLY
WITH THE COMMUNITY THROUGH THE
DS CONNECT PLATFORM AND THEN I
WILL SAY TO MISS ROAN AND MISS
LOMBARDO, THANK YOU FOR SHARING.
AND ISAIAH, THANK YOU FOR BEING
WITH US AND SHARING YOUR STORY
SO WELL AND AUTHENTICALLY.
>> THANK YOU.
>> I SUPER APPRECIATE IT.
I HOPE YOU GET GREAT TIME WITH
YOUR FRIENDS, AS MUCH AS YOU CAN
OUTSIDE OF WORK.
>> YES, YES.
THANK YOU.
>> FANTASTIC.
THANK YOU ALL SO MUCH FOR
JOINING US.
WE REALLY APPRECIATE IT.
>> THANKS FOR HAVING US.
>> THANK YOU.
>> I'LL TURN IT BACK OVER TO DR.
RASOOLY.
>> FIVE CONCEPTS FOR YOUR
CONSIDERATION NOW.
FOR EACH CONCEPT, I'LL ASK THE
STAFF TO PROVIDE A BRIEF
OVERVIEW OF THE INITIATIVE AND
ASK COUNCIL FOR DISCUSSION
REGARDING THE CONCEPT.
AT THE CONCLUSION OF DISCUSSION
I'LL ASK COUNCIL MEMBERS WHETHER
THEY CONCUR OR NOT WITH THE
PROPOSED INITIATIVE.
JUST TO REMIND THE COUNCIL
MEMBERS, CONCEPTS REPRESENT
EARLY PLANNING STAGES FOR
FUNDING INITIATIVES, SUCH AS
REQUESTS FOR APPLICATIONS,
CONTRACT SOLICITATION.
ACCORDING TO LAW, EXPERTS IN THE
FIELD WHICH ARE YOU, THE COUNCIL
MEASURES, MUST APPROVE A CONCEPT
BEFORE IT CAN BE ANNOUNCED OR
DEVELOPED INTO A FUNDING
INITIATIVE.
COUNCIL MEMBERS ARE ASKED TO
REVIEW AND COMMENT ON AND
APPROVE THE CONCEPT'S SCIENTIFIC
MERIT AND PRIORITY.
PLEASE REMEMBER NOT ALL CONCEPTS
ARE DEVELOPED INTO FORMAL
INITIATIVES.
EVEN IF THE CONCEPT LEADS TO
INITIATIVE DETAILS MAY DIFFER
FROM THE ORIGINAL PROPOSAL.
THEY ARE PRESENTED IN OPEN
SESSION, DO NOT HAVE DETAILS,
THERE'S NO, SO TO SPEAK, INSIDE
INFORMATION.
COUNCIL MEMBERS ARE PERMITTED TO
EVEN APPLY FOR FUTURE
INITIATIVE.
HAVING SAID THAT, LET'S HEAR
FROM DR. PILEGGI, NATIONAL
PEDIATRIC MEDICAL DEVICE
ECOSYSTEM.
>> THANK YOU.
PROGRAM SEEKS COUNCIL APPROVAL
FOR INITIATIVE, NATIONAL
PEDIATRIC MEDICAL DEVICE
ECOSYSTEM, MEDICAL TECHNOLOGIES
ARE TRANSFORMING HEALTH CARE,
DRIVING WIDESPREAD ADOPTION OF
DEVICES TO IMPROVE ACCESS TO
CARE, DIAGNOSIS, MONITORING AND
TREATMENT LEADING TO ENHANCED
CLINICAL OUTCOMES, QUALITY OF
LIFE, REDUCED HEALTHCARE COSTS.
THE NUMBERS OF MEDICAL DEVICES
ARE GROWING.
PEDIATRIC LABELING REMAINS
STAGNANT, DESPITE DEMAND,
LIMITED SOLUTIONS ARE AVAILABLE.
OR ADULT DEVICES USED OFF LABEL
DISPOSING CHILDREN.
MED TECH INNOVATION IS GIVEN BY
SMALL BUSINESSES VULNERABLE TO
FAILURE DUE TO REGULATORY
PATHWAYS AND MARKET ENTRY.
PEDIATRIC DEVICE DEVELOPMENT IS
PERCEIVED AS HIGH RISK EFFORT
WITH LOW AND UNCLEAR RETURN ON
INVESTMENT.
INNOVATION IS HINDERED BY LACK
OF INFRASTRUCTURE TO SUPPORT
PEDIATRIC SPECIFIC DEVICE
DEVELOPMENT, VALIDATION,
REGULATORY APPROVAL,
REIMBURSEMENT, THIS WILL ENABLE
NICHD AND NIH LEADERSHIP IN
IMPLEMENTATION OF
MULTI-STAKEHOLDER ENDEAVOR TO
STRENGTHEN THE MEDICAL DEVICE
ECOSYSTEM, ENHANCING
AVAILABILITY OF PEDIATRIC SAFE
MEDICAL DEVICES, IMPROVE
LONG-TERM HEALTH OUTCOMES FOR
AMERICAN CHILDREN, ALIGNS WITH
NIH AND HHS PRIORITIES,
EXECUTIVE ORDER ESTABLISHING
PRESIDENT'S MAKE AMERICA HEALTHY
AGAIN COMMISSION, AND NICHD
STRATEGIC PLAN 2025 FOSTERING
SAFE AND EFFECTIVE THERAPEUTICS
FOR PREGNANT WOMEN, LACTATING
WOMEN, CHILDREN, AND PEOPLE WITH
DISABILITIES.
THANK YOU.
>> THANK YOU.
ARE THERE ANY QUESTIONS OR
COMMENTS ABOUT THIS CONCEPT?
>> THANK YOU.
THANKS FOR THE CONCEPT.
THANK YOU, REBEKAH, FOR THE
INSTRUCTIONS.
I WONDER, IS THERE A ROLE TO
ALSO STUDY DEVICES IN PREGNANCY,
SIMILAR ISSUES AND USING DEVICES
THAT ARE APPROVED, HAVE NORMS
FOR PREGNANT WOMEN, MIGHT BE A
NICE ADDITIONAL PIECE TO ADD, I
WONDER IF YOU COULD COMMENT ON
THAT.
>> SO, THIS SPECIFIC CONCEPT
FALLS IN THE PEDIATRIC
APPLICATIONS.
AS YOU KNOW, PEDIATRIC IS FROM
DAY ZERO THROUGH THE DAY BEFORE
THE 22nd BIRTHDAY BASED ON HOW
FDA REGULATES MEDICAL DEVICES.
THERE IS DEFINITELY VALUE IN
EXTENDING THE DEVELOPMENTAL
DEVICES TO EARLIER, DURING
PREGNANCY, BEYOND THE SCOPE OF
THIS INITIATIVE BUT DEFINITELY
AN AREA WHERE WE'RE VERY
INTERESTED AN MAYBE DR. CERNICH
CAN CHIME IN.
>> WHAT WE'VE TRIED TO DO IS
WE'VE DONE INITIATIVES RECENTLY,
BOTH IN POSTPARTUM PHASE BUT
ALSO WE'VE DONE SOME WORK FOR
STILL BIRTH PREVENTION IN
PREGNANCY, AND REGULAR WORK IN
NATOLOGY DEVICE, SBIR, STTR, WE
HAVE THAT IN THE PORTFOLIO AND
CAN TAKE THE NOTE THAT WE SHOULD
CONSIDER HOW THIS INTERACTS WITH
THE PREGNANCY PERIOD PRIOR TO
PEDIATRICS.
>> THANK YOU.
>> ANY OTHER COMMENTS,
QUESTIONS?
HEARING NONE, LET'S HAVE A
MOTION FOR APPROVAL OF THIS
CONCEPT.
>> MOTION TO APPROVE.
>> AND A SECOND?
>> SECOND.
>> THANK YOU VERY MUCH.
REMEMBER, I'M GOING TO ASK YOU
TO VOTE BY RAISING YOUR HAND.
ALL IN FAVOR?
OKAY.
I SEE EVERYONE'S VOTED IN FAVOR
SO I WON'T ASK FOR OPPOSED OR
ABSTENTIONS.
WE'LL MOVE NOW TO THE NEXT
CONCEPT.
THANK YOU.
DR. KAREN WINIER, PEDIATRIC
GROWTH AND NUTRITION BRANCH,
PEDIATRIC SCIENTIST DEVELOPMENT
PROGRAM.
DR. WINIER?
>> THANK YOU.
THE PEDIATRIC GROWTH AND
NUTRITION BRANCH SEEKS COUNCIL
APPROVAL FOR RENEWAL OF THE
PEDIATRIC SCIENTIST DEVELOPMENT
PROGRAM, AN NICHD-FUNDED
MENTORED TRAINING PROGRAM FOR
PEDIATRICIANS.
THE PROGRAM IS DESIGNED TO
ENHANCE RESEARCH SKILLS AND
PROVIDE A BRIDGE BETWEEN FORMAL
RESEARCH TRAINING AND RECEIPT OF
INDEPENDENT GRANTS.
FIRST ESTABLISHED IN RESPONSE TO
CONCERNS EXPRESSED BY PEDIATRIC
LEADERSHIP THAT ACADEMIC
PEDIATRICSES WERE NOT TRAINED TO
COMPETE FOR NIH GRANTS.
AS MODERN TECHNOLOGY EVOLVED,
THIS PROGRAM CONTINUES TO
FULFILL AND ONGOING NEED IN
TODAY'S HIGHLY COMPETITIVE
RESEARCH ENVIRONMENT.
THIS PROGRAM PROVIDES
PEDIATRICIANS WHO ARE IN THE
EARLY STAGES OF THEIR CAREERS
WITH STATE-OF-THE-ART TRAINING,
EDUCATION, AND MENTORING.
THE PSDP RECRUITS PEDIATRICIANS
IN THE RESIDENCY, TO PARTICIPATE
IN THIS PROGRAM DURING
SUBSPECIALTY FELLOWSHIP
TRAINING.
OVER THE DURATION OF THE
PROGRAM, THE PSDP ADDED
SUBSTANTIALLY TO THE
PHYSICIAN-SCIENTIST WORK FORCE,
THERE IS BEEN A TWO-FOLD
INCREASE IN SCHOLAR APPLICANTS
IN THE PAST FIVE YEARS.
SINCE ITS INCEPTION, THE PSDP
FUNDED 235 PEDIATRICIAN SCHOLARS
LOCATED IN 48 PEDIATRIC
DEPARTMENTS, MENTORED BY
ESTABLISHED SENIOR INVESTIGATORS
WHO SUPPORT SCHOLAR PROJECTS
RELEVANT TO THE ADVANCEMENTS OF
CHILD HEALTH.
MAJORITY OF PAST SCHOLARS HAVE
BEEN NIH-FUNDED PRINCIPAL
INVESTIGATORS, 90% REMAIN IN
ACADEMIC PEDIATRICIAN, MANY HAVE
ANSWERED THROUGH ACADEMIC LADDER
AND HOLD LEADERSHIP POSITIONS.
THANK YOU.
>> THANK YOU.
ARE THERE COMMENTS OR QUESTIONS
ABOUT THIS INITIATIVE?
DR. MALDONADO?
>> THANK YOU SO MUCH.
THIS IS A REALLY WONDERFUL
CONCEPT BUT I GUESS I'M
CONFUSED.
TELL ME ABOUT HOW THIS IS COMING
ABOUT GIVEN THAT WE UNDERSTAND
THAT THIS PROGRAM IS RECENTLY
TERMINATED
>> ORDER TO RECOMPETE ANYTHING,
IN ORDER TO PUBLISH ANY KIND OF
FUNDING OPPORTUNITY, THERE HAS
TO BE A VALID CONCEPT CLEARANCE,
CONCEPT CLEARANCES ARE VALID FOR
FIVE YEARS.
>> NEW CONCEPT THEN OR -- AGAIN,
STILL CONFUSED.
>> SO THE OLD ONE IS GONE.
THIS IS A NEW CONCEPT.
>> IF A PROGRAM IS TERMINATED
THERE HAS TO BE A NEW CONCEPT?
OKAY.
>> THANKS.
>> THAT'S NOT TRUE.
TAKE THAT BACK.
REBEKAH IS SHAKING HER HEAD.
LET'S PUT IT THIS WAY.
WE BRINGING THIS FORWARD AS A
CONCEPT FOR YOUR CONSIDERATION.
HOW ABOUT THAT?
>> OKAY.
>> I GOT A NOD FOR THAT SO
THAT'S WHAT WE'RE GOING WITH.
>> MAYBE I COULD BE A LITTLE
LESS OPAQUE.
>> THANK YOU.
>> WHEN A GRANT IS TERMINATED,
THE GRANT IS NO LONGER ELIGIBLE
TO RECOMPETE.
OR TO BE REMEDIATED IN ANY WAY.
HOWEVER, THAT DOESN'T NEGATE THE
UNDERLYING MOTIVATION OF THE
WORK THAT WE'RE PROPOSING TO DO,
THE CONCEPT.
AND SO IF WE WANT TO RECOMPETE,
IF WE WANT TO COMPETE THIS AND
CONTINUE THIS PROGRAM PERHAPS
SLIGHTLY DIFFERENT FASHION, WE
MUST HAVE A VALID CONCEPT IN
ORDER TO DO THAT.
>> YES.
>> DOES THAT MAKE SENSE NOW?
>> YEAH, I JUST THINK IT WOULD
BE HELPFUL TO GIVE US MORE THAN
THE STANDARD LANGUAGE.
WE'RE ENTERING CONFUSING TIMES.
THE CLEARER YOU CAN BE THE
BETTER WE'RE ABLE TO HELP YOU
ALONG.
THAT'S GOING TO HAPPEN WITH THE
NEXT ONE TOO.
>> I NOTICED IN THE PRESENTATION
AND IN THE COUNCIL WEBSITE THAT
WAS FOCUSED ON MODERN BIOLOGICAL
MEDICINE TECHNOLOGY, NOW PERHAPS
SUGGEST THAT BROADENED
ESPECIALLY AFTER THE
PRESENTATIONS TODAY, ON THE
IMPORTANCE ON DATA SCIENCE.
I KEPT THINKING HOW TRAINING IN
THOSE AREAS REALLY IS IMPORTANT,
IN ANY CAREER DEVELOPMENT OR
OTHER TRAINING PROGRAMS.
>> OKAY.
THAT NOTE IS TAKEN.
I THINK DR. MALDONADO'S POINT,
WE'RE TRYING TO BE AS CLEAR AS
WE CAN WITH RESPECT TO NEED TO
BRING THESE CONCEPTS BACK.
AND SO I THINK WHAT WE WILL BE
ASKING THE COUNCIL FOR IS YOUR
UNDERSTANDING THAT WE'RE
PRESENTING THESE CONCEPTS SO
THEY CAN BE RECOMPETED.
>> THAT'S HELPFUL.
YEAH, I'M TRYING TO UNDERSTAND
PROCESS MORE THAN ANYTHING ELSE.
SO THAT'S FINE.
THANK YOU.
I APPRECIATE HOW HARD YOU'RE ALL
WORKING RIGHT NOW.
JUST WANT TO MAKE SURE I
UNDERSTAND WHAT I'M VOTING FOR.
>> CERTAINLY.
AND SO I THINK WHAT WE'RE DOING
AT THIS MOMENT IS VERY MUCH IN
LINE WITH TRYING TO MAKE SURE
THAT THE PROGRAMS THAT WE KNOW
ARE VERY IMPORTANT FOR THE AREAS
OF SCIENCE THAT ARE IN OUR
MISSION ARE ABLE TO BE
RECOMPETED AND AVAILABLE TO THE
FIELD.
>> DR. AIZER?
>> THANK YOU.
I DON'T KNOW IF THIS QUESTION IS
TOO SPECIFIC FOR THE CONCEPT
PHASE.
BUT HERE IT IS.
TWO PARTS.
FIRST IS ARE THERE AREAS OF THE
COUNTRY THAT ARE UNDERSERVED BY
PEDIATRIC RESEARCHERS, ARE THERE
AREAS WHERE WE HAVE A DEARTH OF
SCHOLARS IN THE SPACE?
AND IF SO, WOULD THIS PROGRAM
SEEK TO ADDRESS THAT BY
CONSIDERING GEOGRAPHIC
DISTRIBUTION IN EITHER
SOLICITATION OR AWARD PHASE?
>> THIS PROGRAM IS DESIGNED SO
THAT ANYONE THAT'S INTERESTED IN
GOING INTO ACADEMIC PEDIATRICS
CAN APPLY.
SO FROM ANY ACADEMIC CENTER IN
THE UNITED STATES.
SO, THEIR PARTICULAR ACADEMIC
CENTER DOESN'T HAVE TO BE
NECESSARILY PART OF A PROGRAM
BEFORE BEING ABLE TO APPLY.
SO THEORETICALLY THE SCHOLARS
CAN COME FROM ANY ACADEMIC
PEDIATRIC CENTER IN THE UNITED
STATES.
>> YES, OF COURSE.
MY QUESTION WAS MORE, YOU KNOW,
IN CONSIDERING AWARDS WILL
GEOGRAPHIC DISTRIBUTION BE
CONSIDERED?
OR WILL THERE BE AN OFFER TO
SOLICIT FOLKS FROM UNDERSERVED
AREAS, OR NOT?
THAT'S FINE.
JUST A QUESTION.
>> WHAT WE CAN DO IN THIS
SITUATION LET US LOOK AT THE
TERMS.
WE CAN TAKE THE NOTE THAT
THERE'S A QUESTION ABOUT
UNDERSERVED AREAS FOR PEDIATRIC
MEDICINE.
AND LET US TAKE THE NOTE AND
BRING THAT BACK FOR
CONSIDERATION AS WE DEVELOP THE
CONCEPT.
>> DR. JABS?
DR. JABS?
>> HI.
I WAS JUST WONDERING WHAT THE
MEANING OF THE TERM BASIC
SCIENCE TECHNOLOGY IS?
IS THAT BROAD ENOUGH?
DOES THAT INCLUDE ALL OF THE
CLINICAL RESEARCH, CLINICAL
TRIALS, ET CETERA, ET CETERA?
>> YEAH, WELL, ACTUALLY I WAS
GIVING SOME OF THE HISTORY, THAT
THAT IS WHAT REALLY THE PURPOSE
WAS WHEN IT FIRST WAS INITIATED.
AND IT WAS -- THE MAIN FOCUS WAS
ON TRANSLATIONAL RESEARCH, IN
OTHER WORDS GIVING THE SCHOLARS
THE BASIS IN WHICH THEY CAN
CREATE VERY INTERESTING -- IF
YOU LOOK AT REALLY THE PROGRAM
ITSELF, RIGHT NOW, IT DOES
INCLUDE -- IT'S VERY WIDESPREAD,
THE SCIENCE IS EXTREMELY
WIDESPREAD.
IT'S NOT JUST FOCUSED IN THE
LAB.
SO, I DIDN'T MEAN TO SAY THAT
THAT WAS JUST THAT AREA OF
TRAINING.
>> SO THE INTENTION IS TO KEEP
IT BROAD.
>> YES.
>> IN ITS CONCEPT.
THANK YOU.
>> ARE THERE ANY OTHER COMMENTS
OR QUESTIONS?
WE CAN ASK FOR A MOTION TO
APPROVE.
>> SO MOVED.
>> THAT WAS --
>> MALDONADO.
>> SECOND?
>> SECOND.
>> ALL RIGHT.
ALL IN FAVOR PLEASE RAISE YOUR
HAND?
THANK YOU SO MUCH.
NOW WE'RE GOING TO MOVE ON AND
HEAR FROM DR. SONIA LEE, HIV
PEDIATRIC, ADOLESCENT, AND
MATERNAL CLINICAL TRIALS.
>> THANKS, DR. RASOOLY.
SEEKS COUNCIL APPROVAL FOR THE
INITIATIVE.
MATERNAL AND PEDIATRIC
INFECTIOUS DISEASE BRANCH
DEVELOPS, IMPLEMENTS DIRECTS
WIDE RANGE OF DOMESTIC AND
INTERNATIONAL RESEARCH
ACTIVITIES FOR THE STUDY OF THE
EPIDEMIOLOGY, DIAGNOSIS,
CLINICAL MANIFESTATIONS,
PATHOGENESIS, TRANSMISSION,
PREVENTION, AND TREATMENT OF HIV
AND ITS COMPLICATIONS, INFANTS,
CHILDREN, ADOLESCENTS, PREGNANT
WOMEN.
BECAUSE OF THE UNIQUE ASPECTS OF
HIV IN PEDIATRIC AND MATERNAL
POPULATIONS, A SPECIFIC CLINICAL
TRIALS NETWORK IS NEEDED TO
DEVELOP AND CONDUCT INNOVATIVE
RESEARCH WITH FOCUS IMPACT ON
IMPROVING LONG-TERM HEALTH OF
POPULATIONS AFFECTED BY AND
LIVING WITH HIV.
NOTABLE ACCOMPLISHMENTS INCLUDED
REDUCTION OF VERTICAL HIV
TRANSMISSION TO LESS THAN 2%,
APPROVAL TO TREAT INFANTS AND
CHILDREN, APPROVAL OF
PRE-EXPOSURE PROPHYLAXIS FOR HIV
PREVENTION FOR ADOLESCENTS.
THIS NETWORK WILL ENCOMPASS
APPROACHES TO ADDRESSING ONGOING
PUBLIC HEALTH ISSUES WITH
INVESTIGATIVE LEADERSHIP,
OPERATIONS AND DATA
COORDINATION, COST
EFFECTIVENESS, CLINICAL TRIALS
SITE CONSORTIUMS, THE
INFRASTRUCTURE SUPPORTED BY THIS
NETWORK WILL ALSO ENGAGE IN
COLLABORATIONS AND STUDIES WITH
OTHER HIV AND RELEVANT
INFECTIOUS DISEASE CLINICAL
STUDIES AS WELL AS FORGE NEW
PARTNERSHIPS, ASSURING
IMPROVEMENT OF HEALTH OF ALL
PEDIATRIC AND MATERNAL HIV
POPULATIONS.
THIS PROPOSED CONCEPT ALIGNS
WITH NICHD STRATEGIC PLAN AND
2025 AND 2025 POST AND COST
CUTTING AREAS.
IT ALIGNS WITH NICHD PRIORITIES
AND THOSE OF THE OFFICE OF AIDS
RESEARCH.
THANK YOU.
>> ARE THERE ANY QUESTIONS OR
COMMENTS ON THIS INITIATIVE?
DR. MALDONADO?
>> YES, THANK YOU FOR THAT
DESCRIPTION.
WILL THERE BE POTENTIAL FOR
GLOBAL INVOLVEMENT AS WELL FOR
THESE, FOR THIS CONCEPT DOWN THE
LINE?
>> YES.
AND WE'LL MAKE SURE THE
INITIATIVE IS ALIGNED WITH ALL
OF OUR NIH POLICIES AND DOING
GLOBAL AND FOREIGN
COLLABORATIONS.
SO YES.
>> OTHER QUESTIONS OR COMMENTS?
SEEING NONE, THEN IS THERE A
MOTION TO APPROVE THIS CONCEPT?
>> SO MOVED, BONNIE MALDONADO.
>> AND A SECOND?
>> SECOND.
>> THANK YOU VERY MUCH.
ALL IN FAVOR RAISE YOUR HAND.
OKAY.
THANKS VERY MUCH.
LET'S MOVE ON TO HEAR FROM DR.
VIRGINIA SALO, CHILD DEVELOPMENT
AND BEHAVIOR BRANCH, ABOUT THE
IMPACT OF TECHNOLOGY AND DIGITAL
MEDIA EXPOSURE USAGE ON CHILD
AND ADOLESCENT DEVELOPMENT.
>> THANK YOU.
PROGRAM SEEKS COUNCIL APPROVAL
FOR INITIATIVE, IMPACT OF
TECHNOLOGY AND DIGITAL MEDIA
EXPOSURE AND USE ON CHILD AND
ADOLESCENT DEVELOPMENT.
TECHNOLOGY AND DIGITAL MEDIA,
TDM, HAVE BECOME INTEGRAL PART
OF DAILY LIVES OF CHILDREN AND
FAMILIES.
MOST CHILDREN NOW ENGAGED
STARTING FROM EARLY AGE WITH
HIGH LEVELS OF EXPOSURE ACROSS
MULTIPLE DEVICES AND WIDE RANGE
OF CONTENT, URGENT NEED TO
UNDERSTAND IMPACT OF DEVELOPMENT
AND INTERACTION BETWEEN FAMILY
MEMBERS, PEERS, SOCIETY.
IN THE FIRST CYCLE NICHD
SUPPORTED THREE AWARDS THAT HAVE
FOCUSED ON BUILDING FOUNDATIONAL
KNOWLEDGE.
THIS INVESTMENT HAS PROVIDED
EXEMPLARS WITH METHODOLOGICAL
RIGOROUS RESEARCH TO ADVANCE
SCIENTIFIC KNOWLEDGE BASE BUT
ALONG WITH TDM WORKSHOP SURFACED
GAPS AND CHALLENGES THAT MUST
ENABLE PROGRESS GOING FORWARD.
THE GOAL IS TO SUPPORT
INNOVATIVE AND TRANSDISCIPLINARY
RESEARCH.
OUR APPROACH INVOLVES BALANCING
RISK, DESIGNING FLEXIBILITIES TO
PURSUE NOVEL TECHNOLOGIES AND
MEASUREMENT TO APPROACHES.
THE CONCEPT TOPIC CUTS ACROSS
RESEARCH PORTFOLIOS WITHIN CHILD
DEVELOPMENT AND BEHAVIOR BRANCH,
ALIGNED WITH STATED EMPHASIS, TO
BE BROADLY INCLUSIVE OF NICHD
PRIORITY POPULATIONS.
THIS ALIGNS WITH NICHD'S 2025
STRATEGIC PLAN THEME FOCUSED ON
IMPROVING CHILD AND ADOLESCENT
HEALTH AND EMPHASIS ON
CAPITALIZING ON ARTIFICIAL
INTELLIGENCE TO IMPROVE HEALTH
AND BIRTH OUTCOMES.
IT WILL PROVIDE OPPORTUNITIES TO
ADVANCE TRANSDISCIPLINARY AND
CROSS-CUTTING SCIENTIFIC TOPICS,
OPPORTUNITIES FOR PROJECT
EMBEDDED CAREER ENHANCING
OPPORTUNITIES TO SUPPORT THE
NEXT GENERATION OF SCIENTISTS.
>> THANK YOU SO MUCH.
ARE THERE ANY QUESTIONS OR
COMMENTS?
NOT SEEING ANY, DO I HAVE A
MOTION TO APPROVE THIS CONCEPT?
>> I CAN MOVE TO APPROVE.
>> AND SECOND?
SECOND.
SECOND, DAMIEN.
>> THANK YOU SO MUCH.
ALL IN FAVOR?
ALL RIGHT.
EXCELLENT.
AND FINALLY WE'LL HEAR FROM DR.
O'REILLY, PRESENTING ClinGen
GENOMIC CURATION EXPERT PANELS.
>> BEFORE YOU BEGIN, I BELIEVE
DR. JABS AND DR. VANDVIDER SAID
THEY WOULD NOT BE VOTING ON THIS
INITIATIVE.
>> THANK YOU.
PROGRAM SEEKS COUNCIL APPROVAL
FOR REISSUE OF THIS INITIATIVE,
ClinGen GENOMIC CURATION
PANELS, SUPPORTING STIMULATION
OF EXPERT PANELS THAT MANUALLY
CURATE, REVIEW, DEFINE CLINICAL
RELEVANCE OF GENES AND VARIANTS
AS PART OF ClinGen.
AS TESTING BECOMES MORE
PREVALENT, UTILIZED BY PATIENTS
AND PROVIDERS, THERE ARE MORE
IDENTIFIED VARIANTS WITH UNKNOWN
SIGNIFICANCE AND EVEN GREATER
NEED TO ASSESS IMPACT ON
DISEASES.
EXPERT PANELS ARE REQUIRED TO
UTILIZE FDA APROFIT ClinGen
INFRASTRUCTURE INCLUDING DATA
STANDARDS AS THEY EVALUATE AND
SHARE DATA LINKING GENOMICS TO
DISEASES; PROVIDING FOCUSED ON
SUPPORT A CURATION,
PARTICIPATING PARS, CONTINUING
LEADERSHIP IN IMPORTANT DOMAIN
AND CONTINUE PROMOTING CURATION
EFFORTS IN GENES AND CONDITIONS
RELEVANT TO OUR MISSION AND
POPULATIONS OF INTEREST.
NOTABLY, APPLICATIONS ARE
ENCOURAGED RELATED TO PRIORITY
AREAS FOR EIGHT NICHD BRANCHES,
AND PAR ALIGNS WITH ALL OF NICHD
STRATEGIC POLLS.
OVER THE PRIOR THREE
OPPORTUNITIES NIH RECEIVED 68
APPLICATIONS, NICHD SPECIFICALLY
FUNDED 10 OF THE 22 AWARDS
ACROSS NIH SUPPORTED 31 EXPERT
PANELS.
71 PUBLICATIONS CITE, HELPING
CURATE OVER 800 GENES AND 3800
VARIANTS.
ULTIMATELY THESE EXPERT PANELS
CONTRIBUTE AUTHORITATIVE
CENTRALIZED PUBLIC RESOURCE
ABOUT AGAINST CONDITIONS AND
CLINICALLY RELEVANT GENETIC
VARIANTS THAT CAUSE THEM.
>> THANK YOU SO MUCH.
ARE THERE ANY COMMENTS OR
QUESTIONS ON THIS INITIATIVE?
I DON'T SEE ANY COMMENTS OR
QUESTIONS, ANY HANDS.
ALL RIGHT.
CAN WE HAVE A MOTION TO APPROVE
THIS ONE?
>> I MOVE TO APPROVE, BONNIE
MALDONADO.
>> A SECOND?
>> SECOND.
CHANCEY.
>> ALL RIGHT.
THANK YOU SO MUCH.
ALL IN FAVOR?
OKAY.
THANK YOU ALL.
THANKS TO MY COLLEAGUES FOR
THESE INTERESTING CONCEPTS.
AND NOW WE'LL MOVE TO ANOTHER
PIECE OF BUSINESS.
EACH YEAR WE ASK FOR APPROVAL OF
COUNCIL STATEMENT OF
UNDERSTANDING.
WHICH OUTLINES THE GENERAL
PROCEDURES TO BE USED FOR SECOND
LEVEL REVIEW.
THE STATEMENT ADDRESSES COUNCIL
MEMBERSHIP STRUCTURE, PROCEDURES
TO REVIEW GRANT APPLICATIONS
DURING CLOSED SESSION,
ADMINISTRATIVE DECISIONS,
ACTIONS THAT DO NOT REQUIRE
COUNCIL RECOMMENDATION, VOTING
OPTIONS, EXPEDITED REVIEW,
PROCEDURES, CONCEPT CLEARANCE,
EMERGENCY PROCEDURES.
WE'VE UPDATED STATEMENT OF
UNDERSTANDING TO REFLECT
NIH-WIDE POLICY CHANGES WITH
RESPECT TO APPLICATIONS.
PLEASE NOTE THIS HAS NOTHING TO
DO WITH THE EARLIER DISCUSSION
OF HOW WE'RE CHANGING THE
APPLICATIONS.
THIS HAS TO DO WITH A DIFFERENT
POLICY CHANGE.
BEGINNING WITH THE NEXT ROUND OF
APPLICATIONS FOR THE NEXT
COUNCIL MEETING, WE HAVE TO
TREAT ANY FOREIGN PRINCIPAL
INVESTIGATOR, EVEN IF IT'S JUST
ONE PRINCIPAL INVESTIGATOR AMONG
SEVERAL, AND EVEN IF THE AWARD
IS GOING TO U.S. INSTITUTIONS,
WE HAVE TO TREAT THOSE
APPLICATIONS AS A FOREIGN
APPLICATIONS.
WE'RE PROPOSING EDITS TO
STATEMENT OF UNDERSTANDING IN
THE SECTION ON ACTIONS THAT MAY
BE CONSIDERED INDIVIDUALLY BY
COUNCIL.
DO WE HAVE A SLIDE FOR THIS?
I THOUGHT WE MIGHT.
OKAY, WE DON'T.
ALL RIGHT.
SO IN THE STATEMENT OF
UNDERSTANDING WHERE IT SAYS THAT
COUNCIL WILL CONSIDER
APPLICATIONS INDIVIDUALLY WHEN
THEY ARE WITHIN A FUNDABLE RANGE
FROM FOREIGN INSTITUTIONS, WE'RE
SUGGESTING ADDING THE WORDS "OR
WHERE ONE OR MORE PRINCIPAL
INVESTIGATORS ON MULTIPLE P.I.
APPLICATION IS AFFILIATED WITH A
FOREIGN INSTITUTION."
SO CONSIDER THE APPLICATIONS
INDIVIDUALLY WHEN FROM A FOREIGN
INSTITUTION OR WHEN ONE OR MORE
P.I.s IS FROM A FOREIGN
INSTITUTION.
THAT'S ONE CHANGE.
AND THEN SIMILARLY COUNCIL WILL
CONSIDER INDIVIDUALLY REQUESTS
TO TRANSFER AN EXISTING AWARD TO
A FOREIGN INSTITUTION OR BETWEEN
FOREIGN INSTITUTIONS, AND WE'RE
ADDING THE LANGUAGE "OR ADDING A
FOREIGN PRINCIPAL INVESTIGATOR
ON A MULTIPLE P.I. APPLICATION."
THOSE ARE THE TWO PROPOSED
CHANGES.
ARE THERE ANY QUESTIONS WITH
RESPECT TO THE PROPOSED
STATEMENT OF UNDERSTANDING AND
THESE TWO PROPOSED CHANGES?
DR. MALDONADO?
>> THANK YOU.
ONE QUESTION.
I HAVE TWO.
FIRST ONE IS, IF YOU HAVE A U.S.
INVESTIGATOR WHO ALSO HAS AN
AFFILIATION WITH FOREIGN
INSTITUTION AS WELL, WOULD THAT
PERSON THEN COUNT AS A FOREIGN
INVESTIGATOR?
WE FREQUENTLY GET APPLICATIONS
FROM PEOPLE WITH JOINT
APPOINTMENTS AT AN INTERNATIONAL
SITE.
JUST WONDERING WHAT THAT -- I
DON'T WANT TO MAKE IT DIFFICULT
BUT THIS IS GOING TO COME UP FOR
SURE.
>> THAT'S THE PROBLEM WITH EVERY
POLICY WE MAKE.
ALL PEOPLE OUT THERE ALWAYS
FIGURE OUT THINGS WE NEVER
THOUGHT OF.
I MUST TELL YOU THAT'S SOMETHING
THAT DID NOT COME UP IN ANY OF
THE MYRIAD -- LET ME SAY
TYPICALLY WHEN PEOPLE APPLY FOR
AWARDS THIS LIST AN AFFILIATION,
REGARDLESS OF WHAT THE OTHER
APPOINTMENTS ARE.
THAT'S MY EXPERIENCE.
AND SO IF YOU ARE APPLYING FROM
UNIVERSITY OF OKLAHOMA, THE
MONEY WILL BE YOU IN OKLAHOMA
IT'S NOT FOREIGN REGARDLESS OF
WHETHER YOU HAVE A LECTURESHIP
IN NORWAY OR SOMEWHERE ELSE.
>> THANK YOU.
THIS FOREIGN INSTITUTION
COMPONENT, LET'S SAY YOU DO HAVE
THAT, DOES IT FALL INTO THE NEW
POLICY?
DOES IT KICK INTO A NEW PROCESS
OR JUST A WHOLE SEPARATE -- I
MEAN, IS IT JUST TO BE NOTED?
WILL THEY BE HANDLING
DIFFERENTLY.
>> EXCELLENT QUESTIONS.
I ACTUALLY HAVE NO IDEA.
WHAT CAN I SAY?
WE HAVE NOT SEEN THE NEW
PROCESS.
WE DO NOT KNOW WHAT IT LOOKS
LIKE.
>> GOT IT.
SO THIS IS REALLY TO ALIGN WITH
THE NEW PROCESS.
>> THE PRINCIPLE IS CLEAR.
IF THE APPLICATION THAT WE'RE
CONSIDERING TO PAY HAS A FOREIGN
PRINCIPAL INVESTIGATOR, THEN
COUNCIL MUST CONSIDER IT
INDIVIDUALLY.
>> I SEE.
OKAY.
>> IF THE CHANGE IS WITH RESPECT
TO FUNDING FOREIGN SITES, SUCH
THAT THAT SITUATION WILL NEVER
EVER ARISE AGAIN, THEN YOU WILL
NOT SEE IT BEING BROUGHT TO YOU
IN THE COUNCIL.
>> GOT IT.
OKAY.
THANK YOU.
>> DR. JABS?
>> MY QUESTION IS, IS THIS FOR
NICHD OR I.C.?
WE'RE TRYING -- EACH COUNCIL IS
BEING ASKED THIS, IS THAT
CORRECT?
>> THAT IS TRUE.
>> I SEE.
I UNDERSTAND NOW.
OKAY.
SO THIS IS BEING PUT FORTH AS AN
OVERALL NIH, AND THEN WE AS A
COUNCIL MEMBER CAN VOTE OUT, FOR
THIS, YEAH, REPRESENTING THIS
INSTITUTE.
THANK YOU.
>> CAN YOU QUALIFYING THE FORUM
FOR THE COUNCIL?
>> SO, FORUM IS MORE THAN
HALF -- QUORUM IS MORE THAN HALF
OF THE APPOINTED MEMBERS, AND WE
NOW HAVE EIGHT APPOINTED
MEMBERS.
AND SO QUORUM WOULD BE FIVE.
AND FORTUNATELY WE HAVE SEVEN ON
THE LINE SO WE'RE IN VERY GOOD
SHAPE.
DO I HAVE A MOTION TO APPROVAL
THE MOTION --
>> I HAVE ANOTHER QUESTION.
SO SORRY.
I TAKE THESE VOTES AS WE ALL DO
SERIOUSLY.
WHAT HAPPENS IF THIS DOES NOT
PASS BY COUNCIL, WHAT WOULD
HAPPEN?
>> WE NEED TO HAVE A VALID
STATEMENT OF UNDERSTANDING THAT
WE RENEW EACH YEAR.
>> OKAY.
>> WE WOULD HAVE TO KEEP GOING
AT IT UNTIL WE GOT YOU TO
APPROVE SOMETHING THAT YOU COULD
ALL APPROVE AND THAT MIGHT MEAN
WE WOULD HAVE TO PUBLISH AND
HOLD ANOTHER PUBLIC MEETING TO
DO THAT BETWEEN NOW AND
SEPTEMBER.
WHICH IS I'M SURE SOMETHING
EVERYONE WOULD LOOK FORWARD TO.
>> BUT MY QUESTION WAS IF THIS
IS NIH-WIDE AND THERE ARE
MULTIPLE COUNCILS VOTING ON
THIS, DO OUR VOTES COUNT IF
EVERYBODY ELSE -- HOW IS IT
PROPORTIONED OUT OR DOES EACH
COUNCIL HAVE TO HAVE A MAJORITY
THOUGH?
>> IT'S LIKE A TWO-PAGE
DOCUMENT THAT LISTS HOW WE WORK
WITH THE COUNCIL.
WE SAY, ALL RIGHT, WE'RE GOING
TO BRING THE FOLLOWING
INDIVIDUAL ACTIONS TO YOU WHEN
WE COME UP, WE'LL BRING YOU
APPEALS, CERTAIN KINDS OF
SPECIAL COUNCIL REVIEW AND
CERTAIN KINDS OF THINGS.
THERE'S A WHOLE LIST THERE.
IT LISTS HOW WE HOLD MEETINGS.
IT WILL LIST -- EXPLAINS HOW WE
WOULD DO EXPEDITED REVIEW.
THERE'S AN ENTIRE DISCUSSION OF
OUR PROCESS.
EACH COUNCIL HAS A DIFFERENT
STATEMENT OF UNDERSTANDING.
ALL THAT WE ARE ASKING IS TO
RENEW THE EXISTING STATEMENT OF
UNDERSTANDING WITH THESE TWO
LANGUAGE CHANGES SO THAT WE
COMPLY WITH A NEW NIH-WIDE
POLICY.
IT IS NOT AS IF THIS IS THE ONLY
QUESTION ON WHICH YOU'RE BEING
ASKED TO VOTE, TO ENDORSE THIS
POLICY OR NOT ENDORSE THIS
POLICY.
IT'S SIMPLY TO MAKE SURE THAT WE
HAVE A STATEMENT OF
UNDERSTANDING OF HOW, WHAT RULES
GOVERN HOW COUNCIL OPERATES.
I HOPE THIS IS HELPING.
THIS WOULD HAVE BEEN IMPROVED BY
A SLIDE.
>> I CAN PUT THE LANGUAGE IN IF
THAT'S HELPFUL, DR. AIZER.
WELL ACTUALLY, IN THE CHAT.
>> JUST QUICKLY, BASED ON PAST
EXPERIENCE, ABOUT HOW MANY MORE,
YOU KNOW, CASES WOULD THE
COUNCIL BE ASKED TO REVIEW WITH
THIS NEW CHANGE?
>> I HAVE NO IDEA.
RIGHT?
BECAUSE THIS NEW FOREIGN AWARD
POLICY IS GOING TO COME AND
SEPARATE OUT THE FOREIGN
COMPONENTS I THINK OF ANY AWARD
SO I DON'T REALLY KNOW.
IT MAY BE ZERO.
REMEMBER, YOU WOULD ONLY
CONSIDER THEM INDIVIDUALLY IF WE
WERE PROPOSING THEM FOR FUNDING.
THERE'S LOTS OF THINGS THAT WE
GET FROM FOREIGN INSTITUTIONS
WHERE THE SCORES ARE NOT
MERITORIOUS.
ALL RIGHT.
CAN I HAVE A MOTION TO APPROVE
THE 2025 PROPOSED STATEMENT OF
UNDERSTANDING?
>> MOVE TO APPROVE.
>> AND A SECOND?
>> SECOND.
CHANCEY.
>> THANK YOU.
ALL IN FAVOR PLEASE VOTE.
ALL RIGHT.
WELL, ANY OPPOSED?
PLEASE RAISE YOUR HAND.
SO, LET ME -- WAIT.
LET'S START AGAIN.
DR. JABS, YOU VOTED, WAS THAT IN
FAVOR OR OPPOSED?
ALL OPPOSED PLEASE RAISE YOUR
HAND.
I DON'T SEE ANY VOTES OPPOSING.
IS ANYONE SAYING THEY ARE
ABSTAINING?
OKAY.
ALL RIGHT.
IT SEEMS LIKE THE PROPOSED
STATEMENT OF UNDERSTANDING HAS
NOW BEEN APPROVED.
DR. CERNICH?
>> AS ALWAYS, THANK YOU FOR
LEADING THE DIFFICULT POLICY
DISCUSSIONS.
I APOLOGIZE FOR MY INARTFL
CONTRIBUTIONS.
THANK YOU FOR JOINING US FOR THE
OPEN SESSION.
I WANT TO RECOGNIZE THIS IS THE
LAST COUNCIL MEETING DR.
CONTRERAS WILL SERVE AS LIAISON.
PEPE, WE ARE GRATEFUL FOR YOUR
SERVICE AND WOULD LIKE TO OFFER
A FEW MINUTES FOR COMMENTS.
PEPE, ARE YOU STILL WITH US?
HE MAY HAVE DROPPED OFF.
OKAY.
WELL, THANKING HIM FOR HIS
SERVICE REMAINS.
I'LL SHARE PEPE ALSO SPURRED
SOME INTEREST FOR MY YOUNGER
DAUGHTER IN NEUROSCIENCE WITH A
PRESENTATION, SMITHSONIAN, ON
ELECTRICAL STIMULATION AND ART
WHEN SHE WAS 5 OR 6.
I'VE BEEN GRATEFUL FOR THE
INTERSECTION OF HIS WORK IN
NEUROSCIENCE AND WITH THE ARTS
AND WILLINGNESS TO COME FORWARD
AND DO PUBLIC PRESENTATIONS OF
THAT, THAT ARE REALLY
ACCESSIBLE.
SO, THIS CONCLUDES THE FIRST DAY
OF OUR TWO-DAY NICHD ADVISORY
COUNCIL MEETING.
I THANK YOU ALL FOR ATTENDING
AND LOOK FORWARD TO SEEING
EVERYONE TOMORROW AT 9:00 A.M.
FOR CLOSED SESSION.
START TIME WILL BE 9:00 A.M.
EASTERN STANDARD TIME.
THANK YOU FOR THE INDULGENCE OF
THOSE ON THE WEST COAST FOR THE
EARLY START.
HAVE A GREAT EVENING, EVERYONE.
>> THANK YOU, SEE YOU TOMORROW.