>>WELCOME TO
THE 180th MEETING OF THE NICHD
ADVISORY COUNCIL.
ONCE AGAIN WE APPRECIATE YOUR
COMBINATION OF THE VISUAL
FORMAT.
TODAY THE ENTIRE MEETING IS OPEN
TO THE PUBLIC AND BEING
BROADCAST ON THE NIH'S VIDEOCAST
NETWORK.
I'D ALSO LIKE TO THANK THE
MEMBERS OF THE PUBLIC WHO ARE
PRESENT ON THIS CALL ESPECIALLY
THOSE WHO ARE REPRESENTING
PROFESSIONAL SOCIETIES AND
RESEARCH ORGANIZATIONS NATURAL
TO EVERYONE WHO IS ABLE TO JOIN
US TODAY.
WITH THE VIRTUAL MEETING FORMAT,
PLEASE REMEMBER TO UNMUTE
YOURSELF BEFORE SPEAKING SO THE
TRANSCRIBER AND VIDEOCAST
OBSERVERS WILL KNOW WHO IS
SPEAKING.
AFTER YOU SPEAK, PLEASE BE SURE
TO MUTE YOUR MICROPHONE IN ORDER
TO PREVENT FEEDBACK LOOPS OR
OTHER AUDITORY INTERFERENCES.
I'M SURE EVERYONE IS FAMILIAR
WITH THIS AFTER TWO YEARS ON
ZOOM.
WITH US FOR THE FIRST TIME AS
REGULAR MEMBERS OF THE COUNCIL
I'D LIKE TO WELCOME DR.
DR. MOLDONADO AND I'LL LIKE TO
CALL ON HOME TO BRIEFLY
INTRODUCE THEMSELVES.
LET'S START WITH DR. MALDONADO.
>> I'M PROFESSOR OF GLOBAL
HEALTH AND INFECTIOUS DISEASES
IN THE DEPARTMENT OF PEDIATRICS
AT STANFORD UNIVERSITY AND HAVE
A POINT APPOINTMENT IN
POPULATION HEALTH.
MY BACKGROUND IS A PEDIATRIC
INFECTIOUS DISEASE
EPIDEMIOLOGIST ON STUDIES OF
REAL WORLD VACCINE EFFECTIVENESS
IN PRIMARY INDIGENOUS
POPULATIONS AND IN POPULATIONS
OF CHILDREN WITH HIV INFECTION.
MY MAIN AREAS OF INTEREST HAVE
BEEN POLIO VACCINE
EFFECTIVENESS, ORAL POLIO
VACCINE TRANSMISSION AND
MODELLING OF TRANSMISSION
DYNAMICS OF O.P.B. TO SIMULATE
THE VACCINE TRANSMISSION IN
REAL-WORLD SETTINGS AND INFANT
AND MOTHER INFECTIONS.
>> THANK YOU FOR JOINING US AND
UNFORTUNATELY YOUR EXPERTISE IN
POLIO VIRUS IS GOING TO BE
NEEDED AGAIN.
IS DR. BAIER WITH US?
I DON'T SEE HIM ON OUR SCREEN
BUT PLEASE SPEAK UP IF YOU ARE.
NOW I'LL TURN THE MEETING OVER
TO DR. REBEKAH RASOOLY IN OUR
DIVISION OF EXTRAMURAL
ACTIVITIES.
>> THANK YOU AND WELCOME.
GOOD AFTERNOON.
I'M GOING TO REVIEW
CONFIDENTIALITY AND CONFLICT OF
INTEREST FOR YOU.
ON THE COUNCIL WEBSITE YOU
SHOULD HAVE AGREED AND SIGNED
AND READ THE CONFIDENTIALITY AND
NON-DISCLOSURE RULES FOR SPECIAL
GOVERNMENT EMPLOYEES BEFORE YOU
EVALUATED ANY OF THE GRANT
APPLICATIONS.
AS SO, YOU SHOULD HAVE RESER
RESERVRESERVICE
-- RECEIVED YOUR CONFLICT OF
INTEREST FORM AND IF NOT PLEASE
LET US KNOW TO SEND THE
APPROPRIATE DOCUMENTS.
PLEASE SIGN THE CERTIFICATION
FORM PRIOR TO THE CLOSED SESSION
TOMORROW AND SEND THEM IN.
ALSO REMEMBER YOU CANNOT SERVE
ON ANY NIH PEER-REVIEW PANEL
WHILE SERVING AS A COUNCIL
MEMBER AND YOU CANNOT PROVIDE
SECOND-LEVEL REVIEW OF COUNCIL
FOR ANY APPLICATION FOR WHERE
YOU PARTICIPATED IN THE FIRST
VIEW.
IT'S NIH'S POLICY AN INDIVIDUAL
CANNOT SERVE ON FIRST AND SECOND
LEVELS OF PEER REVIEW.
IN ADDITION I WANT TO BRING TO
YOUR ATTENTION DURING THE CLOSED
SESSION, PLEASE SHUT OFF
CLOUD-BASED VOICE SERVICES SUCH
AS ALEXA OR OTHER DEVICES AS
THESE DEVICES MAY BE ABLE TO
CAPTURE CONFIDENTIAL
INFORMATION.
THANK YOU.
LET'S THEN MOVE ON TO OUR FIRST
ORDER OF BUSINESS WHICH IS TO
APPROVE THE MINUTES FROM THE
LAST MEETING ON JUNE 13 AND 14,
2022.
THESE MINUTES HAVE BEEN POSTED
ON THE COUNCIL MEMBER WEBSITE
AND ONCE THEY ARE APPROVED, WILL
BE POSTED ON THE NICHD PUBLIC
COUNCIL SITE.
THIS IS THE FIRST SITUATION IN
WHICH YOU'LL BE VOTING AND LET
ME REMIND YOU THE WAY YOU DO
VOTING IS GO TO THE BOTTOM OF
YOUR SCREEN AND YOU SEE THE
BUTTON CALLED REACTIONS AND WHEN
YOU PRESS ON THAT YOU'LL SEE
THERE'S A GREEN CHECK ARROW FOR
YES OR RED X FOR NO AND IF
YOU'RE AABSTAINING --
ABSTAINING JUST RAISE YOUR HAND
AND WE'LL TABULATE YOUR VOTES.
MAY HAVE A NOTION APPROVE THE
JUNE MINUTES.
>> THIS IS SHERRI BARKIN.
MOVE TO APPROVE.
CAN SOMEONE ENDORSE THAT MOTION.
>> THIS IS CATHERINE.
I SECOND.
>> PLEASE VOTE FOR OR AGAINST OR
ABSTAIN USING THE BUTTONS ON THE
BOTTOM OF YOUR ZOOM SCREEN.
WE'LL THEN MOVE FORWARD.
WE'RE GOING TO BE MEETING AGAIN
AS WE DO THREE TIMES A YEAR.
NEXT ON JANUARY 24, 25.
AS WE WILL DO --
>> EXCUSE ME, REBEKAH, I THINK
DR. EGIN HAD A QUESTION?
>> NO, I DIDN'T HAVE A QUESTION.
I COULDN'T VOTE BECAUSE I WASN'T
THERE IN JUNE SO I THOUGHT
RAISING MY HAND WAS TO SAY
ABSTAIN.
I'M SORRY.
>> THANK YOU.
>> SO WE MEET JANUARY 24 AND
25th A VIRTUAL MEETING AND THE
JANUARY MEETING WILL BE A
VIRTUAL MEETING BECAUSE OF THE
DIFFICULTIES OF TRAVELLING IN
THE WINTER.
FOLLOWING THAT, SUBSEQUENT
MEETING IS JUNE 6, 7, WE HOPE
WE'LL BE IN PERSON AND THE REST
OF THE DATES GOING FORWARD ARE
SEPTEMBER 6 AND 7, JANUARY 22
AND 23, 20 AND SEPTEMBER 4 AND
5, 2024.
I'LL GIVE THE MEETING BACK TO
DR. BIANCHI FOR THE DIRECTOR'S
REPORT.
>> THANK YOU, REBEKAH.
I'LL WAIT FOR THE SLIDES TO COME
UP.
>> IT'S HARD TO BELIEVE BUT IT'S
SEPTEMBER AND WE'RE HERE FOR
OUGHT MANY COUNCIL MEETING.
SO TODAY WE HAVE QUITE A NUMBER
OF TOPICS TO COVER.
I'M HOPING TO SPEND ABOUT 30
MINUTES ON THIS MORE FORMAL
PRESENTATION AND THEN LEAVE 15
MINUTES FOR QUESTIONS.
SO WE'LL TALK ABOUT THE 60th
ANNIVERSARY BRIEFLY.
OVER ALL NIH LEADERSHIP UPDATES,
THE BUDGET WHICH IS ALWAYS A
SOURCE OF GREAT INTEREST AND
UPDATES ON THE NICHD RACIAL AND
ETHNIC EQUITY PLAN, BRIEF UPDATE
ON RECOVER FOR THE PEDIATRIC
POPULATION OR LONG COVID AND
UPDATE ON THE IMPROVE INITIATIVE
WHICH HAS BEEN OUR MAIN FOCUS
OVER THE LAST FEW MONTHS ON
MATERNAL HEALTH RESEARCH AND I
THOUGHT I WOULD PRESENT TO YOU A
SUCCESSFUL OUTCOME FROM A DATA
SET THAT HAS BEEN DEPOSITED IN
OUR DATA AND SPECIMEN HUB AND
THEN SINCE WE WILL BE SEGUEING
INTO INTRAMURAL RESEARCH I DID
WANT TO TELL YOU ABOUT THE
CLINICAL CENTER PEDIATRIC
RESEARCH STRATEGIC PLAN AND THEN
END WITH NICHD STAFF UPDATES.
SO IN ABOUT A MONTH WE WILL BE
CELEBRATING NICHD'S 60th
ANNIVERSARY ON THE EXACT DAY
THAT NICHD WAS OFFICIALLY
RECOGNIZED OCTOBER 17, 1962 AND
ORGANIZED AROUND OUR MISSION
WHICH IS HEALTHY PREGNANCIES,
HEALTHY CHILDREN AND HEALTHY AND
OPTIMAL LIVES.
IF YOU HAVEN'T ALREADY
REGISTERED, WE CERTAINLY WELCOME
TO YOU ATTEND VIRTUALLY AND THE
LINK IS HERE.
WE ALSO HAVE AN ACTIVELY UPDATED
ANNIVERSARY WEB PAGE AND THAT
HAS BEEN GIVING US KEY ADVANCES
AND MILESTONES IN NICHD'S
HISTORY.
IT'S ALSO HAD A SERIES OF
PROFILES ON PEOPLE WHO HELP
NICHD ACHIEVE ITS MISSION.
AND IT WILL ALSO TALK A LITTLE
BIT ABOUT NICHD'S FUTURE
RESEARCH DIRECTIONS.
SO AS A SAID, IT'S AN ACTIVE
LIVING WEB PAGE THAT IS
FREQUENTLY UPDATED SO WE
ENCOURAGE YOU TO GO THERE AND
MORE IMPORTANTLY WE HOPE YOU CAN
JOIN US FOR PART OF THE
ANNIVERSARY SYMPOSIUM.
SO I THINK MOST OF YOU KNOW THAT
DR. FRANCES COLLINS WHO SERVED
AS THE DIRECTOR OF NIH FOR 12
YEARS, A RECORD-SETTING AMOUNT
OF TIME AND SERVING MULTIPLE
PRESIDENTS AND STEPPED DOWN AS
THE DIRECTOR OF NICHD IN
DECEMBER 2021.
DR. LARRY TABAK WHO WAS
PRINCIPLE DEPUTY DIRECTOR FOR
DR. COLLINS INITIALLY BECAME THE
ACTING DIRECTOR.
WHY DOES HE HAVE THIS STRANGE
TITLE, PERFORMING THE DUTIES OF
THE NIH DIRECTOR.
THAT'S BECAUSE THERE'S A TIME
LIMIT ON HOW LONG IN AN ACTING
POSITION AT NIH.
THE GOVERNMENT HAS ITS WAYS AND
SO NOW HIS TITLE HAS CHANGED TO
PERFORMING THE DUTIES OF THE NIH
DIRECTOR.
WHAT THAT MEANS IS HE IS
EFFECTIVELY THE ACTING NIH
DIRECTOR.
THE WHITE HOUSE CONTINUES TO
CONDUCT A SEARCH FOR THE NEXT
NIH DIRECTOR.
NOW, REMEMBER THIS IS A
POLITICAL APPOINTMENT INITIALLY
MADE BY THE PRESIDENT AND THEN
REQUIRES SENATE CONFIRMATION.
SO WHOMEVER IS APPOINTED WILL
ONLY HAVE TWO CONFIRMED YEARS
PENDING WHOEVER IS THE NEXT
PRESIDENT OR PRESIDENT BIDEN
CONTINUES IN HIS ROLE.
DR. TABAK HAS INFORMED US THAT
THEY REALLY DON'T EXPECT THAT
ANYONE WILL BE NAMED UNTIL
DECEMBER AT THE EARLIEST.
SO HE'S REALLY GONE FROM AN
ACTING, I'M NOT GOING TO MAKE A
DECISION MODE TO NOW REALLY
SAYING WE NEED TO MOVE FORWARD
AND MAKE ACTIVE DECISIONS
PARTICULARLY ON A NUMBER OF THE
HIGH-LEVEL SEARCHS THAT ARE
ONGOING AS FOR THE DIRECTOR OF
NCATS IS OPEN.
WE HAD HAD AN APPOINTMENT FOR
THE NEW DIRECTOR OF THE NATIONAL
CANCER INSTITUTE.
DR. BOTONOLI ALSO A POLITICAL
APPOINTMENT AND FOR THOSE WHO
DON'T KNOW THERE'S REALLY ONLY
TWO LEADERSHIP APPOINTMENTS AT
NIH THAT ARE POLITICAL.
ONE IS THE OVER ALL DIRECTOR AND
ONE IS THE DIRECTOR OF THE
NATIONAL CANCER INSTITUTE.
FORTUNATELY FOR DR. BERTAGNOLLI
DOESN'T HAVE GO THROUGH SENATE
CONFIRMATION AND SHE IS AN
ONCOLOGIST AND SURGEON.
SHE'S CURRENTLY PROFESSOR OF
SURGERY AT HARVARD MEDICAL
SCHOOL.
SHE'S A SURGEON AT BRIGHAM AND
WOMEN'S HOSPITALS AND A MEMBER
OF THE SARCOMA DISEASE CENTER AT
THE DANA FARBER CANCER
INSTITUTE.
SHE'S SPEARHEAD PRESIDENT
BIDEN'S CANCER MOON SHOT
INITIATIVE.
PRESIDENT BIDEN IS IN BOSTON
TODAY AT THE J.F.K. LIBRARY
CELEBRATING THE 60th ANNIVERSARY
OF PRESIDENT KENNEDY'S MOON SHOT
INITIATIVE.
IT'S MAKING THE LINK BETWEEN THE
CANCER MOON SHOT AND THE MOON
SHOT TO THE ACTUAL MOON.
DR. BERTAGNOLLI IS IN BOSTON AND
DON'T KNOW IF SHE'LL BE JOINING
FOR THAT EVENT OR NOT BUT I DO
KNOW I'VE ALREADY HAD A LOVELY
COVERING WITH HER PARTICULARLY
BECAUSE SHE IS VERY INTERESTED
IN STREAMLINING CLINIC CARE AND
RESEARCH FOR CHILDREN WITH
CANCER.
I'M EXCITED SHE HAS THIS
INTEREST IN PEDIATRIC CANCER AND
FULLY WANTS TO INCLUDE NICHD IN
THE OVER ALL MOON SHOT
INITIATIVE SO CHILDREN WILL BE
REPRESENTED.
I LOOK FORWARD TO MEETING HER IN
PERSON.
WE ONLY HAVE SPOKEN ON
TELEPHONE.
THE BUDGET AND APPROPRIATION.
FISCAL YEAR '23 BUDGET THAT HAS
BEEN PROPOSED BY THE PRESIDENT
PROPOSED $62.5 BILLION FOR NIH.
IT HAS INCLUDES $1.68 BILLION
FOR NICHD.
IF YOU ARE REALLY PAYING
ATTENTION YOU MIGHT KNOW THAT'S
EXACTLY THE KIND OF BUDGET WE
HAVE IN THE CURRENT FISCAL YEAR
'22 BUDGET.
BECAUSE OF THE WAY THE
PRESIDENT'S BUDGET IS DEVELOPED
AND THE CONGRESSIONAL
JUSTIFICATION, THERE'S
DISCONNECT ON TIMING.
IT DOESN'T MEAN THE BUDGET WILL
STAY FLAT.
IT'S JUST THAT THIS IS WHAT WAS
FORMALLY PUT INTO PLACE AND
EXPECT THE NUMBER WILL BE HIGHER
FOR NICHD.
SEPTEMBER 30th IS A COUPLE WEEKS
AWAY THE END OF OUR PHYSICAL
YEAR.
WE DO NOT HAVE ANY INFORMATION
ABOUT FISCAL YEAR '23.
WE KNOW CONGRESS HAS BEEN
ACTIVELY WORKING ON THE FISCAL
'23 BUDGET BUT IT'S A LONG WAY
FROM BEING FINALIZED.
WE THINK A CONTINUING RESOLUTION
WHEREBY WE CONTINUE WORKING WITH
AN EXPECTED FLAT BUDGET IF WE
GET OUR OTHER BUDGET WILL OCCUR
BUT THERE'S A POSSIBILITY OF A
GOVERNMENT SHUTDOWN BECAUSE THEY
HAVEN'T SIGNED THE CONTINUING
RESOLUTION.
I DON'T THINK ANYONE WANTS THE
GOVERNMENT TO SHUTS DOWN
ESPECIALLY PRIOR TO AN ELECTION
CYC
CYCLE.
I DID WANT TO POINT OUT THIS
YEAR THE BUDGET WAS SIGNED ON
MARCH 15, WHAT THAT MEANS IS WE
REALLY DON'T KNOW HOW MUCH
BUDGET IS IN UNTIL MARCH AND THE
IMPROVE INITIATIVE COULDN'T GET
STARTED UNTIL MARCH UNTIL WE
FOUND OUT WHETHER THE PROGRAM
WILL BE APPROPRIATED.
I THINK THIS IS SOMETHING I
DIDN'T REALLY APPRECIATE UNTIL I
JOINED NIH THE DIFFICULTIES
PROGRAM STAFF HAVE IN TERMS OF
JUGGLING BETWEEN WHAT IS KNOWN
AND WHAT IS NOT KNOWN.
WE REALLY WANT TO GIVE OUR KUDOS
TO ALL OF OUR PROGRAM STAFF AND
GRANTS MANAGEMENT STAFF WHO HAVE
BEEN DEALING WITH THIS VERY
SHORT TIME LINE IN THIS SPECIFIC
FISCAL YEAR.
ANOTHER THING THAT WAS PRESENT
IN THE APPROPRIATIONS LANGUAGE
WAS THAT NICHD WOULD ESTABLISH A
TASK FORCE ON STILLBIRTH IN THE
UNITED STATES.
IT'S STILL A MAJOR PROBLEM AND
AFFECTS 1 IN 160 PREGNANCIES AND
THE RATE OF EARLY STILLBIRTHS IS
STILL UNCHANGED AFTER 30 YEARS.
WE ARE ESTABLISHING SUBCOMMITTEE
OF THE NICHD COUNCIL, THAT'S ALL
OF YOU, AND THOSE THAT WILL BE
ASKED TO PARTICIPATE WILL BE
RECEIVING ALET -- A LETTER SOON
BUT THE MEMBERS WILL ALSO
INCLUDE MEMBERS FROM THE CDC
OTHER INSTITUTES AND CENTERS
OUTSIDE NIH, THOSE IN MATERNAL
MEDICINE AND GENOMICS.
IT'S AN AREA THAT WASN'T REALLY
FLOURISHING WHEN WE HAD OUR
STILLBIRTH TASK FORCE IN THE
PAST AND THE FUNDED INITIATIVE
ON STILLBIRTH.
THIS TIME WE'VE BEEN ASKED TO
FOCUS ON NO-RISK FACTORS FOR
STILLBIRTH, FACTORS IN
COLLECTING DATA ON STILLBIRTHS
IN THE UNITED STATES.
FOCUS ON COMMUNITIES AT HIGHER
RISK ON STILLBIRTH AND THE
IMPACT ON MOTHERS FOLLOWING A
STILLBIRTH.
BASICALLY PEOPLE WILL GET THEIR
LETTERS SOON AND THE MEETINGS
WILL HAVE TO BE SOON AND WE CAN
TALK ABOUT IT IN THE QUESTION
AND ANSWER PERIOD.
NOW I'D LIKE TO MENTION THE
NICHD RACIAL AND EQUITY PLAN
KNOWN AS THE REAP.
THIS IS A MAJOR COMPONENT OF
NIH'S UNITE PROGRAM.
IT'S PART OF THE "I" COMMITTEE.
WHERE THEY WERE U-N-I-T-E AND
THIS IS THE "I" COMMITTEE FOR
ACQUISITION, INCLUSION AND --
EQUITY, INCLUSION AND
EXCELLENCE.
WE RECENTLY HAVE FILLED OUT A
DOCUMENT TO SHOW THE PROGRESS
THAT HAS BEEN MADE IN TERMS OF
THE IMPLEMENTATION PLAN AND THE
REAP ACTIVITIES HAVE HELPED US
IDENTIFY ADDITIONAL TARGETS AND
PROGRAMS AND THIS IS UNIQUE TO
NICHD.
>> HERE ARE SOME OF THE
ACTIVITIES WE'VE BEEN ABLE TO
ACCOMPLISH DURING FISCAL YEAR
'22.
WE CONDUCTED A PULSE SURVEY OF
THE NICHD CLIMATE AMONGST THE
STAFF AND DISSEMINATED THE
RESULTS IN A TOWN HALL MEETING
LAST SPRING.
WE'VE BEEN EXAMINING WORKFORCE
DATA WITH DEMOGRAPHICS, EQUITY
AND INCLUSION ACROSS ALL
POSITIONS AN PAY GRADES AND
PARTICULARLY EXAMINING,
RECRUITMENT, RETENTION AND AWARD
DATA FOR INSTITUTE EMPLOYEES
BASED ON DEMOGRAPHICS.
WE ARE ALSO ESTABLISHING
TRAINING CURRICULA AND
OPPORTUNITIES FOR DEVELOPMENT OF
OUR OWN INSTITUTE PERSONNEL.
AND SOMETHING WE HAD A LOT OF
ABOUT IN THE PULSE SURVEY WAS
PROVIDING OPPORTUNITIES FOR
STAFF TO PROVIDE BASIC
LEADERSHIP ENROLLED WITHIN AND
OUTSIDE THE INSTITUTE IN AN OPEN
AND TRANSPARENT FASHION.
PREVIOUSLY, PEOPLE WERE
RECOMMENDED FOR MEMBERSHIP ON
COMMITTEES OR TRANS-NIH
INITIATIVES AND NO ONE KNEW HOW
THOSE PEOPLE WERE SELECTED.
THERE'S AN OPEN VOLUNTEERING
PROCESS AND EVERYTHING IS
TRANSPARENT WITH AN EYE IN
GIVING OPPORTUNITIES TO
EVERYONE.
THE NUMBERS FOR COVID CASES ARE
COMING DOWN RIGHT NOW.
WE'RE CERTAINLY NOT OUT OF THE
PANDEMIC BUT WE MIGHT HAVE A
LITTLE BIT OF A RESPITE OVER THE
NEXT FEW MONTHS.
NICHD STAFF ARE PARTICIPATING IN
RECOVER WHICH YOU PROBABLY KNOW
IS ESSENTIALLY THE LONG COVID
NIH WIDE INITIATIVE OR COVER
STANDS FOR RESEARCHING COVID TO
ENHANCE RECOVERY.
IT IS REALLY LOOKING AT THE
LONG-TERM HEALTH EFFECTS OF
COVID.
AND IT'S AN OBSERVATIONAL STUDY
WHICH MEANS THE RESEARCHERS HAVE
ED INFORMATION FROM
PARTICIPANTS.
IT IS NOT AN INTERVENTION STUDY.
WE'RE VERY GRATEFUL THE COVID
LEADERSHIP ALLOWED US TO INCLUDE
A WHOLE SEPARATE ARM ON
PEDIATRIC COVID, PREGNANT PEOPLE
ARE BEING INCLUDED IN THE ADULT
INITIATIVE AND WE ARE CONSTANTLY
REMINDING THE RECOVER STAFF THAT
WE WANT TO INCLUDE PEOPLE WITH
DISABILITIES AS WELL.
HERE ARE SOME OF THE STUDY
QUESTIONS AS THEY RELATE TO
CHILDREN WITH LONG COVID.
HOW MANY OF THEM ARE GETTING
LONG COVID, WHY DO SOME GET IT
AND OTHERS DO NOT.
WHAT SYMPTOMS DO CHILDREN FEEL
WHEN THEY GET LONG COVID.
HOW LONG ARE THEY SICK WHEN THEY
GET IT.
WHAT ARE THE UNDERLYING CAUSES
AND HOW DOES LONG COVID AFFECT
LATER PHYSICAL HEALTH, MENTAL
HEALTH AND DEVELOPMENT IN
CHILDREN.
THIS IS PARTICULARLY IN CHILDREN
FOR THE PEOPLE WHO WERE PREGNANT
WITH COVID THIS WILL BE A
MECHANISM TO STUDY THEIR
OFFSPRING IN THE WOMB DURING THE
INFECTION AND HOW DO CONGENITAL
EXPOSURE TO SARS COV2 INFECTION
DURING PREGNANCY AFFECT CHILD
PHYSICAL HEALTH AND CHILD
DEVELOPMENT.
THERE'S A BASELINE TIER THAT
CONSISTS OF 19,500 DIADS.
THERE IS A SECOND TIER WITH
ACUTE FOLLOW-UP AT 2,4 AND 8
WEEKS.
THAT'S AROUND 800 DIADS.
THERE'S A TIER 3, DEEP
PHENOTYPING IN YEARS 1 AND 2.
AROUND 600 AND POST-ACUTE
LONGITUDINAL FOLLOW-UP AT 6, 12,
24, 36 AND 48 MONTHS HOPING TO
RECRUIT 6,000 PARTICIPANTS.
THE STUDY ONLIED TO CREN --
ONLY OPENED IN APRIL AND THE 50%
OF THE SITES WERE ACTIVATED AND
I THINK THERE'S OVER 700 NOW.
THEY HAD QUITE A WAYS TO GO TO
GET 19,500 PARTICIPANTS.
IF YOU GO TO RECOVER COVID.org
YOU CAN GET MORE INFORMATION IF
YOU KNOW ANYONE OF ANY AGE THAT
CAN PARTICIPATE BUT FOR CHILDREN
SPECIFICALLY THERE'S A SEPARATE
ARM FOR CHILD HEALTH AND COVID.
THE IMPROVE INITIATIVE IS
BUILDING ON EFFORTS MULTIPLE
INSTITUTES HAVE MADE SINCE A
MEETING WE HAD WITH THE BLACK
MATERNAL HEALTH CAUCUS IN
DECEMBER OF 2019.
FOR THE FIRST TWO FISCAL YEARS
EACH INSTITUTE AS WELL AS THE
OFFICE OF THE DIRECTOR
CONTRIBUTED FUNDS THAT MAINLY
SUPPORTED SUPPLEMENTS AND NOW
WITH THE NEW APPROPRIATION OF
$30 MILLION AND NICHD'S BASE
BUDGET BEGINNING THIS YEAR WE
WERE ABLE TO DO A LOT MORE.
JUST AS A REMINDER, THE IMPROVE
INITIATIVE IS NIH WIDE.
THE FOCUS IS TO SUPPORT RESEARCH
ON REDUCING PREVENTIBLE CAUSES
OF MATERNAL DEATHS, REDUCING
HEALTH DISPARITIES AND
COMPLICATIONS IN
DISPROPORTIONATELY AFFECTED
POPULATIONS AND IMPROVING HEALTH
OF WOMEN, BEFORE, DURING, AND
IMPORTANTLY THE YEAR AFTER
DELIVERY.
WITH AN ENORMOUS AMOUNT OF
EFFORT, STAFF ACROSS MULTIPLE
INSTITUTES WERE ABLE TO GET THE
FOLLOWING NEW INITIATIVES OFF
THE GROUND AND SPEND THE $30
MILLION.
SO THE NOTICE OF SPECIAL
INTERESTS ON DISSEMINATION AND
IMPLEMENTATION RESEARCH RESULTED
IN MULTIPLE APPLICATIONS THAT
HAVE BEEN REVIEWED AND THERE IS
A FUNDING PLAN IN PLACE NOW.
AND STUDIES OF MATERNAL HEALTH
INTERVENTIONS AND A FOCUS ON
LONGITUDINAL HEALTH RECORD FOR
THE PREGNANT PERSON AND CHILD
AND LINKAGES FOR WOMEN, BEFORE
PREGNANCY AND AFTER PREGNANCY.
AND WE'VE USED NEW OPPORTUNITIES
FOR THE CHALLENGE WHICH I'LL
TALK ABOUT IN A MOMENT.
THE CHALLENGE FUNDING MECHANISMS
AND WHAT WE DECIDED TO DO WAS
DELAY THE MATERNAL HEALTH
CENTERS OF EXCELLENCE TO FISCAL
YEAR '23.
THE GOAL WAS TO USE THE CURRENT
MONEY BECAUSE WE COULD ONLY
ESTABLISH THE INITIATIVES IN
MARCH AND THE NOTICES HAD TO GO
OUT, PEOPLE HAD TO APPLY, THEY
HAD TO BE REVIEWED, FUNDING
PLANS HAD TO BE ESTABLISHED ALL
WITHIN A PERIOD OF ABOUT SIX
MONTHS OR LESS THAN SIX MONTHS.
SO WE DIDN'T WANT TO RUSH THE
MATERNAL HEALTH CENTERS
EXCELLENCE.
ALL THESE INITIATIVES WERE
INTENDED TO GIVE US INFORMATION
THAT THEN COULD BE USED FOR THE
CENTERS OF EXCELLENCE WITH AGAIN
A SPECIFIC EMPHASIS ON
COMMUNITY-BASED EFFORTS TO
ADDRESS LOCAL SOLUTIONS THAT
WOULD HAVE A LOCAL AFFECT
ESPECIALLY ON HEALTH
DISPARITIES.
YOU PROBABLY HAVEN'T HEARD WE
TALK ABOUT CHALLENGES AND WE
SUCCESSFULLY COMPLETED OUR FIRST
CHALLENGE THIS FISCAL YEAR
THOUGH NIH CONDUCTED OVER ALL 62
CHALLENGES OVER THE PAST DECADE
OR SO.
.
WHAT ARE CHALLENGES?
THEY'RE A WAY FOR FEDERAL
AGENCIES TO CROWD SOURCE AND
ENGAGE PUBLIC INNOVATORS
DIRECTLY TO FIND INTERESTING
IDEAS OR SOLUTION TO IMPORTANT
PROBLEMS.
THEY'RE NOT THE TYPICAL ACADEMIC
INVESTIGATORS.
THIS IS JUST PUBLIC SO ANYBODY
CAN RESPOND AND WE CAN OFFER
MONETARY AND NON-MONETARY
BENEFITS TO COMPETITION
CANDIDATES AND OFFICIALS,
NETWORKING, OPPORTUNITIES TO
SPEAK AT A CONFERENCE TO
HIGHLIGHT THE INNOVATION.
THE BENEFITS TO US OF THE
CHALLENGE INCLUDE THE FOLLOWING,
THERE'S NO OBLIGATION OF AN
AWARD.
WE PAY A PRIZE, ESSENTIALLY FOR
A WINNING SOLUTION OR SOLUTIONS
ONLY.
THERE CAN BE MANY POSSIBLE
SOLUTIONS SO WE CAN ENCOURAGE
CREATIVE SOLUTIONS AND THIS
CREATES A BROADER POOL OF
ENTRANTS TO INCREASE THE NUMBER
AND DIVERSITY SOLVERS AND RAISES
AWARENESS OF A PARTICULAR PUBLIC
HEALTH NEED AND ENCOURAGES
APPLICATION OF EXISTING NIH
RESEARCH DATA TO ADDRESS PUBLIC
HEALTH OR MEDICAL ISSUES.
IT ALSO STIMULATES PUBLIC SECTOR
INVESTMENT.
IT'S CRITICAL FOR US TO ENGAGE
THE COMMUNITY TO IMPROVE
MATERNAL HEALTH.
ONE CHALLENGE FOCUSES ON
ENGAGING THE COMMUNITY.
THIS ENCOURAGING COMMUNITY-BASED
AND ADVOCACY ORGANIZATIONS IN
THE U.S. TO DEVELOP THE
INFRASTRUCTURE AND CAPABILITIES
NECESSARY TO CONDUCT MATERNAL
HEALTH RESEARCH.
INTERESTING, THERE'S NO
REQUIREMENT TO PARTNER WITH AN
ACADEMIC INSTITUTION.
THERE'S A TOTAL PRIZE PURSE OF
$3 MILLION TO BE DISTRIBUTED
ACROSS MULTIPLE PHASES.
THERE'S DIFFERENT PHASES WHERE
GROUP WILL APPLY AND GET
SUBSEQUENT SUPPORT WITH THEIR
APPLICATION AND GET AN INITIAL
PRIZE POOL OF MONEY TO HELP WITH
SUBSEQUENT ASPECTS OF THEIR
APPLICATION AND NON-MONETARY
ASSISTANTS AND WRITING SUPPORT
AND RESEARCH ACTIVITY.
THIS IS WHERE IT'S IMPORTANT
BECAUSE WE WANT TO GET AT THE
GRASSROOTS AND GET DIRECTLY INTO
THE COMMUNITIES THAT MAY NOT
HAVE THE SUPPORT THAT ARE
AVAILABLE IN ACADEMIC
INSTITUTIONS.
HERE'S THE ANNOUNCEMENT.
AND PLEASE GO TO THE
ANNOUNCEMENT AND SUBMISSION
PORTAL.
WE HAVE TWO CHALLENGES AS PART
OF THE IMPROVE INITIATIVE.
THE RADX PORTAL WAS SO
SUCCESSFUL FOR THE DEVELOPMENT
OF TESTING.
MOST DON'T RECOGNIZE WHEN YOU DO
A SELF-TEST FOR COVID ALMOST ALL
THAT TECHNOLOGY WAS DEVELOPED BY
NIH.
AND NIBIB PLAGE A HUGE ROLE IN
IMPLEMENTING THAT TECHNOLOGY.
WE THOUGHT IT WAS SO SUCCESSFUL
IN THE WAY IT WAS ORGANIZED AND
FELT IT WOULD ADDRESS A
PARTICULAR CHALLENGE WE HAD WITH
WOMEN IN THE SO-CALLED MATERNITY
CARE DESERTS WHO ARE IN REGIONS
THAT LACK ACCESS TO MATERNAL
CARE.
HOW CAN YOU MONITOR THESE PEOPLE
WHO ARE PREGNANT WHO DON'T HAVE
ACCESS TO CARE AND CONNECT THEM
WITH THE APPROPRIATE SPECIALIST.
THE MATERNAL HEALTH CHALLENGE
PRIORITIZES HOME-BASED OR POINT
OF CARE WEARABLES LIKE A FIT BIT
AND OTHER DEVICES AND OTHER
REMOTE SENSING TECHNOLOGIES TO
EXTEND THE POSTPARTUM CARE AND
THERE WERE CONCERNS IN TERMS OF
APPROVAL AND CAN HAVE AN IMPACT
ON POSTPARTUM MYOPATHY FOR
PEOPLE IN THE HEALTH DESERTS.
WE HAVE PRIZES AWARDED THROUGH
SEVERAL PHASES AGAIN.
HERE'S THE CHALLENGE
ANNOUNCEMENT.
THIS ONE WILL OPEN THIS WEEK
THERE'S A BIT MORE TIME FOR THE
SUBMISSION DEADLINE IN NOVEMBER.
THE MONEY HAS BEEN SET ASIDE AND
WE'RE ANXIOUS TO AWARD THAT
MONEY.
FOR FISCAL YEAR '23 THE VISION
IS TO FUND SEVERAL MATERNAL
RESEARCH CENTERS OF EXCELLENCE.
THERE WASN'T ENOUGH TIME TO GET
THE FUNDING OPPORTUNITY
ANNOUNCEMENT OUT AND WE CAN GET
THEM REVIEWED IN THIS FISCAL
YEAR.
WE DIDN'T WANT TO
DISPROPORTIONATELY GIVE AN
ADVANTAGE TO INSTITUTIONS THAT
ARE HEAVILY RESOURCED THAT CAN
MOVE THE NEEDLE VERY QUICKLY.
THAT'S WHY WE DECIDED TO WAIT
UNTIL AND THE GOAL IS TO
SIGNIFICANTLY REDUCE MATERNAL
MORBIDITY AND DECREASE
PREVENTIBLE MORTALITY BY
PARTNERING WITH THE COMMUNITIES
AFFECTED AND INCORPORATING THEIR
NEEDS AND PERSPECTIVES IN THE
MATERNAL HEALTH PERFORMED BY THE
CENTERS OF EXCELLENCE.
THERE'LL BE SEVERAL RESEARCH
CENTERS WITH A COORDINATING HUB
AS WELL AS IMPLEMENTATION
SCIENCE RESOURCE HUB.
WE DO SEE A NETWORK OF MULTIPLE
COMMUNITY SITES.
AND THE RESEARCH PROJECTS WILL
HAVE A TRAINING COMPONENT TO
EXPAND THE CADRE OF RESEARCHERS
IN MATERNAL HEALTH.
WE'LL INSIST ON COMMON DATA
ELEMENTS AND MODELS AND HAVE
STRUCTURES COLLECTED ACROSS THE
STRUCTURES ACROSS THE COMMON
DATA HUB AND THE IMPLEMENTATION
SCIENCE RESEARCH HUB WILL
PROVIDE EXPERTISE TO ALL THE
RESEARCH CENTERS TO BETTER
UNDERSTAND HOW EVIDENCE-BASED
RESEARCH CAN BE TRANSLATED TO
MORE EQUITABLE CARE MODELS.
WE EXPECT THE MAJORITY OF
FUNDING IN FISCAL YEAR 23 WILL
BE CENTERED AROUND THE CENTERS
OF EXCELLENCE.
THE IMPROVE INITIATIVE IS BASED
ON THE $30 MILLION BUDGET BUT IN
NICHD'S BASE BUT THE MONEY IS
DISTRIBUTED ACROSS MULTIPLE
INSTITUTES AND CENTERS.
IT'S NOT THE ONLY MONEY DEVOTED
TO RESEARCH IN MATERNAL HEALTH.
WE HAD OVER $400 MILLION IN
TOTAL IN NIH SPENDING ON
MATERNAL HEALTH.
I WANTED TO REMIND EVERYONE IT'S
A CENTRALIZED RESOURCE FOR
RESEARCHERS TO SHARE
DEIDENTIFIED DATA SETS FROM
STUDIES FUNDED BY NICHD.
IT SERVES AS A PORTAL FOR
REQUESTING BIO SPECIMENS FROM
SELECTED STUDIES YOU CAN SEE
TOPICS INCLUDED IN THE DASH AS
WELL AS CURRENTLY AVAILABLE BIO
SPECIMENS.
THE ULTIMATE GOAL IS TO
ACCELERATE SCIENTIFIC FINDINGS
TO ULTIMATELY IMPROVE HUMAN
HEALTH.
IF YOU TYPE IN THE GOOGLE DASH
NICHD IT WILL COME UP RIGHT
AWAY.
IT'S BEEN ENORMOUSLY SUCCESSFUL
AND A FANTASTIC OPPORTUNITY FOR
TRAINEES TO DEVELOP HYPOTHESES
AND TEST THEM WITH ACTUAL DATA.
I WANTED TO HIGHLIGHT ONE STUDY
THAT'S BEEN OUR MOST REQUESTED
STUDY WHICH IS CONSORTIUM ON
SAFE LABOR OR CSL.
THIS STUDY IN DELIVERIES FROM 19
HOSPITALS ACROSS THE UNITED
STATES OVER THE YEARS 2002 TO
2008.
THEY WERE RELEASED IN 2016.
IT'S OUR MOST POPULAR DATA ABOUT
A THIRD OF REQUESTS ARE ON THE
DATA SET.
FROM 194 TOTAL AND THERE'S BEEN
39 PUBLICATIONS USING SO
SECONDARY ANALYSES.
ALMOST HALF OF ALL THE
PUBLICATIONS ASSOCIATED WITH
DASH REQUESTS ARE COMING FROM
THIS PARTICULAR DATA SET.
THIS JUST SORT OF CAME ACROSS MY
ROUTINE PUB MED SEARCHS.
A NEW PAPER THAT'S JUST OUT WITH
THE TITLE OF MATERNAL AND
NEONATAL OUTCOMES OF LABOR AND
THEY USE MACHINE LEARNING AND
RISK PROGRESSION MODELS ON
OUTCOMES.
THEY USE THESE TO PROMOTE
INDIVIDUALIZED CLINICAL
DECISIONS USING BASELINE AND
LABOR CHARACTERISTICS OF
EDUCATION.
AGAIN, THEY NEVER USED THEIR OWN
PATIENTS.
THIS PARTICULAR STUDY IS FROM
THE MAYO CLINIC.
THEY DID ALL THEIR MODELLING
BASED ON THE DASH DATA SET.
I'D LIKE TO MENTION BECAUSE
SEPTEMBER MEETING IS THE ONE
THAT FOCUSES ON OUR INTRAMURAL
RESEARCH PROGRAM.
WE HAVE INTRAMURAL RESEARCHERS
IN THE DIVISION OF INTRAMURAL
POPULATION HEALTH RESEARCH USING
THE CSL DATA SET AND MACHINE
LEARNING METHODS TO IDENTIFY
RISK FACTORS FOR PROLONGED
SECOND-STAGE LABOR.
I WANTED TO REMIND EVERYONE
ABOUT THE NIH CLINICAL CENTER.
IT'S AN OLDER PHOTOGRAPH SHOWN
HERE.
IT'S IN THE CENTER OF THE NIH
CAMPUS.
IT'S THE LARGEST RESEARCH
HOSPITAL IN THE WORLD.
ANYONE WHO COMES IN IS
PARTICIPATING IN A RESEARCH
PROTOCOL.
THERE'S CURRENTLY ABOUT 1600
ACTIVE RESEARCH STUDIES AND
ABOUT HALF OF THEM ARE ON THE
NATURAL HISTORY OF DISEASE
ESPECIALLY RARE AND OFTEN
GENETIC DISEASES.
MOST OF THE OTHER STUDIES ARE ON
CLINICAL TRIALS OFTEN
FIRST-IN-HUMAN STUDIES DRUGS AND
THERAPIES.
ONE OF THE THINGS I REALLY
APPRECIATE ABOUT THE CLINICAL
CENTER BEING AN INVESTIGATOR
THERE'S OPPORTUNITY FOR RESEARCH
EQUITY.
FOR MANY STUDIES, WE INCLUDING
MINES, WE ARE ABLE TO PAY FOR
TRANSPORTATION AND LODGING COSTS
FOR ANYBODY WHO PARTICIPATES IN
OUR RESEARCH STUDY.
THERE'S NO INFORMATION REQUIRED
FROM HEALTH CARE INSURANCE ALL
THE SERVICES ARE AT NO COST TO
THE PARTICIPANT.
WHAT WE DON'T HAVE IS A
STRATEGIC PLAN FOR PEDIATRIC
RESEARCH AND BEING AND BEING ON
THE GOVERNING BOARD I WAS ASKED
TO CO-LEAD STRATEGIC PLANNING
EFFORT FOR THE EFFORT
PEDIATRIC RESEARCH AND HAVE
PLANNING FOR IS WE HAVE 15
PEOPLE WHO ARE CLINICAL
INVESTIGATORS KNOWLEDGEABLE IN
PEDIATRICS AND WHAT IS CURRENTLY
GOING ON.
THE CURRENT WAY PEDIATRIC
RESEARCH IS DONE IS AN
INDIVIDUAL INVESTIGATOR REQUESTS
RESOURCES.
THERE'S NO OVER ALL STRATEGIC
PLAN.
THERE ARE OVER 250 CREDENTIALS
PEDIATRICIAN WHO'S HAVE
PRIVILEGES AT THE CLINICAL
CENTER.
FAR GREATER THAN ANYONE
RECOGNIZED AND WE ALSO HAVE THE
ACTING DIRECTOR OF THE RESEARCH
AND HAPPENS TO BE A PEDIATRIC
NEUROLOGIST AND WAS CHAIR OF
PEDIATRICS AND THE HOSPITAL'S
CEO AT THE CHILDREN'S UNIVERSITY
HOSPITAL OF RODCHESTER.
IT'S GREAT TO HAVE HER ADVICE.
SHE'S NOT A FORMAL MEMBER OF THE
WORKING GROUP BUT WE'RE REPORT
TO HER AS ONE OF THE PEOPLE.
MY HOPE I SAY THIS EVERY TIME
WE'LL HAVE A PERMANENT
SCIENTIFIC DIRECTOR NAMED SOON.
WE HAD 4 EXCELLENT FINALISTS IN
THE SHORT LIST AND WE'LL HEAR
FROM DR. CHRIS McBAIN DOING AN
OUTSTANDING JOB AS OUR ACTING
SCIENTIFIC DIRECTOR OR THE
DIRECTOR OF THE INTRAMURAL
RESEARCH PROGRAM.
DR. PORTER, I THINK I MENTIONED
LAST TIME REQUIRED AS CLINICAL
DIRECTOR.
SINCE SPRING WE HAVE FORMED A
COMMITTEE AND APPLICATIONS WERE
RECEIVED OVER THE SUMMER AND
BEING REVIEWED BY THE SEARCH
COMMITTEE.
AND I BELIEVE THEY'LL START
INTERVIEWS LATER THIS MONTH.
AND THEN THEY'LL BE RECOMMENDING
THE BEST CANDIDATES FOR MY
CONSIDERATION.
THAT'S ALL MOVING ALONG.
WE'RE LOOKING FOR A CHIEF
DIVERSITY OFFICER AND LOOKING
FOR STAFF IN THE OFFICE OF
HEALTH EQUITY.
WE HAVE THREE BRANCH CHIEF
POSITIONS OPEN INCLUDING THE
CHIEF OF THE MATERNAL AND
INFECTIOUS DISEASE BRANCH AND
FERTILITY AND INFERTILITY
BRANCHES AMONG OTHERS.
I'D LIKE TO THANK DR. LEE FOR
BEING THE ACTING CHIEF OF MPIBB
AND DR. DAN JOHNSON WHO IS THE
TRIPLE THREAT WITH HIS OWN
CONTRACEPTIVE RESEARCH BRANCH
AND DIRECTOR OF TWO INSTITUTES.
WE'RE ALSO LOOKING FOR MULTIPLE
SCIENTIFIC REVIEW PROGRAM
OFFICERS AND PROGRAM ANALYSTS
AND TWO THINGS TO KEEP IN MIND
FOR THOSE WHO ARE LISTENING
VIRTUALLY, WE HAVE MUCH MORE
FLEXIBILITY IN SOME OF THESE
POSITIONS TO WORK FULLY REMOTELY
FROM OTHER PLACES IN THE
DOMESTIC UNITED STATES AND DON'T
FORGET THAT FULLY PAID 12-WEEK
MATERNAL LEAVE OPTION WE HAVE IN
THE FEDERAL GOVERNMENT.
WITH THAT, I BELIEVE I'M AT THE
END.
I THINK IT'S MY THANK YOU SLIDE
AND HAPPY TO TAKE QUESTIONS.
HOW ARE WE DOING WITH TIME?
I MAY ARE RAN OVER A LITTLE BIT.
NO, WE HAVE SO MINUTES FOR
QUESTION.
DAVID.
>> I JUST WANTED TO REFLECT
SUPPORT FOR THE STILLBIRTH
INITIATIVE AS A NEONATOLOGIST WE
RECOGNIZE IT'S ONE OF THE GREAT
TRAGEDIES OF SOCIETY AND EFFORTS
TO IDENTIFY RISK AND DETECT OR
PREVENT STILLBIRTH IS A
TREMENDOUS GOAL.
IT WILL HAVE A LOT OF BENEFICIAL
KNOCK-ON EFFECT AMONG MANY
ISSUES YOU JUST DISCUSSED.
>> THANK YOU, DAVID.
I JUST ATTENDED AT A MEETING IN
SAN DIEGO THIS PAST WEEK AND
BEFORE THAT WAS A WORKSHOP ON
THE FETAL GENOMICS CONSORTIUM
WHICH HAS COME OUT OF THE FUNDED
INITIATIVES WE CO-FUNDED WITH
NHGRI.
PEOPLE ARE SAYING THE
CONNECTIONS WITH THE ANOMALIES
WE'RE SEEING PRENATALLY AND
INCLUDE PEOPLE WITH GENOMIC
EXPERTISE IN THE STILLBIRTH
WORKING GROUP.
>> NO QUESTION JUST IMPRESSED
WITH THE BREADTH AND DEPTH OF
THE INSTITUTE.
BONNIE, I KNOW YOU'VE BEEN
INFORMALLY IN A FEW TIMES.
I THINK THE INITIATIVES FOR
STILL BIRTH ARE CRITICAL MANY OF
US HAVE BEEN WORKING IN THE AREA
OF STILLBIRTH AND UNFORTUNATELY
ONLY RECENTLY RECOGNIZED A MAJOR
PROBLEM AND UNFORTUNATELY AS YOU
POINTED OUT WITH NO CHANGES OVER
THE LAST MANY DECADES NOW.
LOOK FORWARD TO THE OMICS
APPROACH AND HOPEFULLY WILL BE A
KEY AND THE RECOVER INITIATIVE
HAS BEEN WORKING AT THE ADULT
LEVEL BUT MORE IMPORTANTLY NOW
TO TRACK WHAT'S GOING TO HAPPEN
IN CHILDREN OVER THE LONG TERM.
AND FINALLY LOOKING FORWARD TO
SEEING APPLICANTS FOR THE MANY
EXCITING CHIEF POSITIONS TO
REALLY LEAD US INTO THE NEXT SET
OF INITIATIVES AS WE REALLY
START TO UNLOCK SOME OF THE
REALLY IMPORTANT AREAS OF
UNDERSTANDING FUNDAMENTAL BASIC
WORK THAT CAN HELP US UNDERSTAND
THE UNDERLYING GENETIC AND
EPIGENETIC CHANGES THAT MAY
IMPACT OUTCOMES IN CHILDREN.
I THINK WE HAVE SO MUCH MORE
CAPACITY TO DO THAT AND IT'S
IMPORTANT TO HARNESS THAT FOR
CHILDREN AND FOR LIFE COURSE.
THANK YOU.
>> THANK YOU.
YOU CAN COME OFF MUTE.
>> MY VOICE IS A LITTLE CRACKED.
JUST GETTING OVER COVID.
I JUST WANT TO SAY THANK YOU.
THE WORK YOU DESCRIBED IS SO
IMPORTANT AS WE THINK OF THE
FUTURE OF WOMEN'S AND CHILDREN'S
HEALTH AND HAVING PROJECTS
LOOKING AT MATERNAL AS WELL AS
FETAL OUTCOMES SIMULTANEOUSLY IS
SO CRITICAL.
I WANT TO THANK YOU FOR THE
LEADERSHIP AND FOR BEING
INCLUDED.
>> WE HOPE YOU RECOVER
UNEVENTFULLY AND SORRY TO HEAR
ABOUT BEING INFECTED RECENTLY.
I CANNOT EXPLAIN WHAT AN
ENORMOUS EFFORT THIS HAS BEEN
FOR THE ENTIRE STAFF.
THEY'VE REALLY ROLLED UP THEIR
SLEEVES.
EVERYONE FOR THE LAST TWO YEARS
HAS BEEN DOING TWO JOBS
ESSENTIALLY.
THEY'RE REGULAR JOB PLUS THEIR
COVID-RELATED JOB AND ON TOP OF
IT TO ASK EVERYONE TO DO THE
WORK IN AN ACCELERATED TIME
LINE.
THIS REALLY ATTESTS TO THE
DEDICATION OF OUR STAFF NOT ONLY
AT NICHD AND ACROSS NIH.
THE FACT THAT PEOPLE ARE SO
COMMITTED TO IMPROVING HEALTH
PARTICULARLY FOR OUR OPERATIONS.
WE'RE TRYING TO RECOGNIZE
PEOPLE, AWARDS AND FINANCIAL
SUPPORT WHERE APPROPRIATE.
>> LOOKS LIKE WE HAVE A LITTLE
BIT OF TIME BUT I KNOW ALL
INSTITUTIONS ARE REALLY JUST
STARTING TO TRY TO RECOUP FROM
THE AFFECTS OF THE PANDEMIC AND
THERE'S STRESS AND WONDERING
WHAT'S DONE AT THE NIH LEVEL TO
HELP WITH STAFF WELLNESS AND
STRESS RELIEF?
I KNOW THIS HAS BEEN QUITE A
DIFFICULT TIME FOR EVERYBODY AND
WORKING IN MANY WAYS MUCH
HARDER.
THIS ZOOM ERA IS NOT AS
FORGIVING AS IN-PERSON WORK AND
WONDERING WHAT INITIATIVES HAVE
BEEN ALLOWED AT THE WHOLE
INSTITUTIONAL LEVEL.
>> THANK YOU FOR THE QUESTION.
WE CERTAINLY RECOGNIZE THE
MENTAL HEALTH EFFECTS NOT ONLY
OF OUR STAFF BUT ON THEIR
CHILDREN, ON ALL MEMBERS OF
SOCIETY AND EXTRAMURAL RESEARCH
WORKFORCE AS WELL.
WITHIN THE NIH THERE'S BEEN A
NUMBER OF WELLNESS INITIATIVES.
WE HAVE RESOURCES WE POINT
PEOPLE TO.
WHEN WE DO OUR MONTHLY TOWN
HALLS WE ENCOURAGE MENTAL HEALTH
BREAKS.
AT NICHD WE HAVE NO MEETING
WEEKS.
WE DISCOURAGE NON-URGENT E-MAIL
MESSAGES OVER THE WEEKEND.
WE'RE APPROACHING PEOPLE TO GET
OUT AND GET EXERCISE.
THERE'S YOGA ACTIVITIES ACROSS
NIH, VIRTUAL YOGA WE'RE VERY
AWARE OF IT AND CERTAINLY
ENCOURAGING PEOPLE TO TAKE CARE
OF THEIR MENTAL AND PHYSICAL
WELLNESS.
I AGREE, I THINK THE HARDEST
THING FOR MOST PEOPLE IS THAT
THE LINE BETWEEN WORK AND HOME
HAS BEEN BLURRED.
YOU ARE ALMOST WORKING AROUND
THE CLOCK AND I CERTAINLY EXPECT
THAT IN MY POSITION BUT MY
HUSBAND HAS MORE OF A 9:00 TO
5:00 JOB AND SEE IT IN HIM HE
FEELS THE NEED TO GO AROUND THE
CLOCK VIRTUALLY.
THERE'S NO QUESTION THERE'S BEEN
SIGNIFICANT MENTAL HEALTH
AFFECTS AND I'M SURE THAT IS
TRUE IN THE U.K. I'LL TAKE
ADAM'S QUESTION AND THEN WANT TO
GIVE A CHANCE FOR INTRODUCTION.
>> WE MENTION ED BIDEN IS SET T
ANNOUNCE A NEW DIRECTOR.
YOU MENTIONED ARPA-H IN THE
PAST.
ONE QUESTION WE HAD GOTTEN FROM
OUR COMMUNITY IS WHERE DOES
PEDIATRICS FIT IN ARPA-H AND IS
THERE A NARRATIVE FOR PEDIATRICS
AND WHAT CAN WE DO TO ADVOCATE
FOR THE NEW AGENCY THAT PREVIOUS
FAILURES INCLUDE A FORWARD
LOOKING VISION.
>> GREAT QUESTION AND YOU'RE
RIGHT.
THIS NEWS CAME THIS MORNING.
I KNOW SOMEONE IS COVERING A
MEETING FOR ME WITH THE
ASSISTANT SECRETARY.
SHE KNOWS THE PERSON WHO HAS
BEEN NAMED AS THE PERMANENT
DIRECTOR OF ARPA-H WHO HAS
PREVIOUS DARPA EXPERIENCE AS
WELL AS BIO TECH EXPERIENCE.
TO ANSWER YOUR QUESTION ABOUT
OPPORTUNITIES FOR PEDIATRIC
RESEARCH, I THINK THIS IS WHERE
WE'RE EXCITING ABOUT MONICA
BERTAGNOLLI'S PRESENTATION AND
THE FACT THAT SHE AINDEPENDENTLY
REACHED OUT TO ME BEFORE SHE
EVEN STARTED IS ONE CONNECTING
THREAD.
THE FACT THAT DR. SART ICH KNOWS
THE NEW DIRECTOR WILL HELP IN
DIRECT WAYS.
WE'LL USE WHATEVER OPPORTUNITIES
WE HAVE TO INCLUDE THE PEDIATRIC
RESEARCH AGENDA WHETHER IT'S
PEDIATRIC DIABETES OR PEDIATRIC
CANCER.
WE INITIATIVELY TALKED WITH
DR. TARA SCHWETZ, WHO AT THE
TIME WORKING IN THE OSTP AND
WHITE HOUSE WAS WORKING ON THE
INITIAL PLANS FOR ARPA-H AND WE
SUGGESTED MATERNAL HEALTH ISSUES
AS WELL AND ECLAMPSIA AND OTHER
CONDITIONS THAT DO NEED NOVEL
THERAPIES.
WE'RE NOT AT THE TOP OF THE LIST
IN TERMS OF THE PRESIDENT'S
AGENDA BUT WE'RE GOING TO BE
INTEGRATING OUR SELVES WHERE WE
CAN.
SO LET ME INTRODUCE DR. FAIR.
I DON'T KNOW IF YOU HEARD ME BUT
WE'D LIKE YOU TO TELL US BRIEFLY
A BIT ABOUT YOURSELF SO EVERYONE
WILL KNOW WHO YOU ARE.
>> SURE.
>> I'M ROUGH AROUND THE EDGES
TOO.
I JUST TESTED POSITIVE FOR COVID
THIS MORNING.
I'M DAMIEN FAIR PROFESSOR AT THE
UNIVERSITY OF MINNESOTA.
I'M IN PEDIATRIC AND INSTITUTE
OF CHILD DEVELOPMENT AND
DIRECTOR OF A NEW INSTITUTE
CALLED THE MASONIC INSTITUTE FOR
THE DEVELOPING BRAIN WHERE WE
ARE TRYING TO PULL TOGETHER MANY
SILOED UNITED STATES AROUND
MATERNAL AND CHILD HEALTH AND
EDUCATION AND PSYCHOLOGY IN ONE
BUILDING TO SPEED UP THE
TRANSLATION TO REACH THE PEOPLE.
IT'S ONE OF THE REASONS I WAS
FRANTICALLY WRITING DOWN THE NEW
INITIATIVES AROUND THE COMMUNITY
WORK AND EFFORTS AND INVESTMENTS
IT'S ONE OF THE MAJOR CHALLENGES
WE PENNED AS MAKING REAL
PROGRESS ESSENTIALLY WORKING
WITH THE COMMUNITY AND THE IDEA
AND THE WORK SO IT'S AN ALL
HANDS ON DECK AFFAIR AND ONE OF
THE CRITICAL POEPTS.
YOU CAN -- CRITICAL COMPONENTS
IT'S NEW TO ME AND I'M LEARNING.
I APPRECIATE THAT AND HAVE MORE
READING TO DO, I GUESS, FOR THE
NEXT WEEK.
>> WE HOPE YOU FEEL BETTER AS
WELL AND HAVE A RELATIVELY
SMOOTH COURSE OF COVID.
IF YOU NEED FURTHER INFORMATION
WE CAN CONNECT YOU EASILY TO
PROGRAM OFFICIALS WHO CAN GUIDE
YOU THROUGH SOME OF THE STEPS.
ONE OF THE THINGS WE DIDN'T
MENTION WAS AN EXISTING
COMMUNITY EDUCATION PROGRAM RUN
BY NHLBI.
THEY'RE ALSO GETTING FUNDING
FROM THE IMPROV INITIATIVE BUT
THOSE ARE WITH ACADEMIC
PARTNERS.
YOU'RE NOT INCLUDED FROM WORKING
WITH AN ACADEMIC PARTNER IN THE
MATERNAL HEALTH CHALLENGE I
MENTIONED BUT WE REALLY WANTED
TO INCLUDE ORGANIZATIONS THAT
DON'T HAVE THAT SUPPORT.
BUT IF YOU HAD THE SUPPORT,
THAT'S OKAY TOO.
REACH OUT TO YOU GET THE
INFORMATION YOU NEED TO PASS ON
TO THE COMMUNITY ORGANIZATIONS.
I WANT TO PASS ON I HAPPEN TO
SPEAK AT A MEETING IN JULY AND
TOOK A TWO-HOUR CAR RIDE WITH
SOMEONE SINGING YOUR PRAISES.
WE'LL MAKE IT UP QUICKLY WITH
THE BREAK.
WITH THE NEW COUNCIL MEMBERS AS
I SAID A COUPLE TIMES, THE
SEPTEMBER MEETING TYPICALLY
FOCUSES ON NICHD'S INTRAMURAL
PROGRAM AND I'M GOING TO TURN
THE MEETING OVER NOW TO
DR. CHRIS McBAIN WHO IS THE
ACTING SCIENTIFIC DIRECTOR OF
THE INTRAMURAL PROGRAM AND WILL
GIVE THE ANNUAL DIVISION OF
INTRAMURAL RESEARCH REPORT.
CHRIS.
OVER TO YOU.
>> GOOD AFTERNOON, EVERYBODY.
FOR THOSE WHO DON'T KNOW ME I'M
CHRIS McVEIGH.
THE CURRENT DIRECTOR FOR THE
INTRAMURAL PROGRAM AND A
SCIENTIST AND INTERESTED IN THE
DEVELOPMENT OF SMALL NEURAL
CIRCUITS INSIDE THE HIPPOCAMPUS
AND LOOKING AT PLASTICITY AND
TRYING TO SOLVE EVOLUTIONARY
QUESTIONS AT THE MOMENT RELATED
TO LEARNING AND MEMORY AND CELL
TYPE IDENTITY.
WE RUN A NEUROSCIENCE LAB INSIDE
CHILD HEALTH.
WHAT I THOUGHT I'D FOCUS ON ARE
ON FOUR MAIN TOPICS TO GIVE YOU
AN OVERVIEW OF THE INTRAMURAL
PROGRAM AS IT STANDS RIGHT NOW.
SOME BUDGET AND PERSONNEL
UPDATES WE'RE WORKING ON AND I
MIGHT SPEND THE TALK ABOUT
INITIATIVES WE STARTED IN OUR
OFFICE OF EDUCATION AND UPDATES
ON OUR HOWARD UNIVERSITY
PARTNERSHIP.
IF ANYBODY HAS QUESTIONS DURING
THE TALK, FEEL FREE TO INTERRUPT
ME OR WE CAN HOLD CONVERSATION
TO THE END.
I'M HAPPY OTHER WAY BUT I'D
RATHER I WAS ABLE TO ANSWER YOUR
QUESTIONS ON THE FLY.
IN OCTOBER OF 2021 IN THE
DIVISION OF POPULATION HEALTH
RESEARCH WAS FOLDED INTO THE
NIH, NICHD AND OUR DIVISION IS
MADE OF EIGHT DIVISIONS.
THE DEFERRED AND DEVELOPMENT
HEALTH RESEARCH AND
TRANSLATIONAL MEDICINE AND
MOLECULAR AND GENOMIC AND
TRANSLATION IMAGING AND THE
OBSTETRICS AND MATERNAL FETAL
MEDICINE.
THAT'S A LARGE PORTFOLIO OF
SCIENCE.
IT RANKS FROM GENETICS THROUGH
ZEBRAFISH THROUGH NEUROSCIENCE
THROUGH OBSTETRICS.
WITH THE ACQUISITION WE
REPRESENT ABOUT 750 PERSONNEL OF
WHICH WE HAVE 75 P.I.s.
61 ARE TENURED.
WE CURRENTLY HAVE 12 TENURE
TRACK INVESTIGATORS AND I'LL
TALK ABOUT THEM TOMORROW.
WE ALSO HAVE TWO ASSISTANT
PERSONAL INVESTIGATORS AND
PEOPLE WHO ARE BECOME SENIOR
SCIENTISTS EMBEDDED IN
PARTICULAR LABS AND THEY SERVE
SUPERVISORY POSITIONS AND WE
HAVE TRAINEES THAT RANGE FROM
GRADUATE STUDENTS THAT
PARTICIPATE IN OVER 30 GRADUATE
PARTNERSHIP PROGRAMS THAT EXIST
ON THE NIH AND HAVE POST-BAC
LAUREATE GRADUATES BEFORE THEY
DECIDE ON A CAREER AND WE HAVE
POST-DOCTORAL AND CLINIC
FELLOWS.
WE'RE CURRENTLY SEPARATED INTO
DIFFERENT GROUPS AND I'LL SHOW
THE COMPOSITION OF THESE IN THE
NEXT SLIDE.
IN THE POPULATION HEALTH
RESEARCH BRANCH THERE'S PROGRAMS
DIVIDED INTO THREE PROGRAMS AND
ONE PROGRAM.
THE EPIDEMIOLOGY BRANCH,
STATISTICS BRANCH AND SOCIAL
BEHAVIORAL SCIENCE BRANCH,
CON
CONTRACEPTIVE DEVELOPMENT
PROGRAM AND HAVE 69 PROGRAMS AND
SIX ACCREDITED TRAINING MEDICAL
PROGRAMS PEDIATRIC, ADULT AND
ENDOCRINOLOGY AND GIYNECOLOGY AD
REPRODUCTIVE AND PERINATAL
RESEARCH AND OBSTETRICS.
WEIR
WE HAVE REDEFINE THE STRUCTURE
OF OUR INTRAMURAL PROGRAM AND
ALLOWED SCIENTISTS TO
SELF-ASSEMBLE INTO 12
AFFINITY-BASED GROUPS.
THOSE ARE LARGELY BASED ON
AROUND THE TOPICS CAPTURED IN
THE RESEARCH PITFALL AND A FLID
ENTITY AND THEY CAN SWITCH
BETWEEN GROUPS TO SEEK OUT
PARTNERS AND COLLABORATORS AS
THEY'VE SEEN FIT TO FOSTER
COMMUNICATION AROUND AREAS AND
THEMES GIVEN WE HAVE SUCH A
LARGE REPERTOIRE OF SCIENTIFIC
INTERESTS.
WE HAVE GROUPS OVERSEEN BY A
NUMBER ASSOCIATE SCIENTIFIC
DIRECTORS AND SORT OF OVERSEE
THE SCIENTIFIC MANAGEMENT AND
SHARED RESOURCES OF THE BUILDING
AND THINGS LIKE SERVICE
CONTRACTS BUT DON'T OVERSEE
INDIVIDUAL LAB BUDGETS.
BOTH AFFINITY GROUP MEMBERS AND
ASSOCIATE SCIENCE DIRECTORS PLAY
A ROLE IN MENTORING TENURE TRACK
INVESTIGATORS.
ONE OF THE INITIATIVES WE'VE
TAKEN SERIOUSLY IS HAVING
INCOMING TENURE TRACK
INVESTIGATORS IMMEDIATELY
ESTABLISH COMMITTEES AROUND THE
RESEARCH PROGRAM AND THE PRIMARY
MENTOR IS THE ASSOCIATE
SCIENTIFIC DIRECTOR OR THE HEAD
OF THEIR AFFINITY GROUP OR ANY
OTHER PERSON OR THREE OR FOUR
MEMBERS INSIDE OR OUTSIDE THE
I.C.
WE FOUND GETTING THE BALL
ROLLING WHEN IT HITS THE GROUND
HAS BEEN INFLUENTIAL IN GETTING
THEM ESTABLISHED.
AS A SCIENTIFIC DIRECTOR I
WORKED WITH EACH P.I. ON THEIR
BUDGET AND RESOURCES NEEDED.
PART OF OUR RESTRUCTURING ABOUT
A DECADE AGO DID AWAY WITH THE
IMMEDIATE LEVEL WHERE P.I.s HAD
TO GO TO THEIR PROGRAM CHIEF OR
LAB CHIEF TO DISCUSS BUDGET AND
RESOURCES AND CAN WORK DIRECTLY
WITH ME ON THE BUDGET AND
RESOURCES AS THEY SEE FIT.
IN ADDITION TO THE ESDs FOR
DIFFERENT BUILDING AS AND THE
NIH CAMPUS WE HAVE THOSE THAT
MANAGE ADMINISTRATIVE PORTFOLIO
SUCH AS BUDGET AND
ADMINISTRATION AND RECRUITMENT
AND RETENTION.
I'LL TELL YOU A LITTLE BIT ABOUT
THE INITIATIVES WE HAVE
ESTABLISHED OVER THE LAST YEAR
AND CONSIDERING THE LATTER ON
RECRUITMENT AND RETENTION.
WE'VE INTRODUCED TWO POSITIONS
THIS YEAR ONE FOR TRANSLATIONAL
RESEARCH.
THIS IS A MAJOR EFFORT UNDERWAY
TO TRY AND FOSTER AN INTERACTIVE
AND INTERCOLLABORATIVE EFFORT
BETWEEN INSTITUTES AND
INVESTIGATORS.
DANNY PORTER WHO HAS TAKEN ON
THE ROLE AS OUR ASD HAS MADE
GREAT INROADS IN BRINGING
TOGETHER LABS TO BENEFIT FROM
THE INTERACTION.
AS DIANA, MENTIONED WE'RE TAKING
OUR DIVERSITY TASK SERIOUSLY AND
HAVE A NEW SCIENTIFIC DIRECTOR
FOR ADIA.
AND THEY TYPICALLY SPAN ABOUT 12
END GROUPS SOP ARE QUITE LARGE
WITH MORE THAN THAT AND I
RECENTLY MELT -- MET WITH THE
AFFINITY GROUPS AND TALKED ABOUT
WHAT COULD BE DONE BETTER AND
WE'RE AT A POINT WHERE WE
UNDERSTAND WE NEED TO JUGGLE
THIS ORGANIZATION A LITTLE MORE
AND INCORPORATE SOME OF THE
SMALLER AFFINITY GROUPS TO ALLOW
MORE TRANSITIONING AND
VISIBILITY BETWEEN AFFINITY
GROUPS.
FOR THE MOST PART THEY'VE BEEN
INCREDIBLY HELPFUL FOR
SCIENTISTS AND TRAINEE TO
INTERACT WITH PEOPLE DOING
LIKE-MINDED SCIENCE BUT IN SOME
WAYS BEING AN INSTITUTE WITH A
VERY BROAD RANGE OF SCIENCE
THESE ARE HELPED FOSTER AN
EDUCATION OF WHAT SCIENCE WE'RE
DOING INSIDE THE DIR AS WELL AS
FOSTERING COLLABORATIONS BETWEEN
THE DIFFERENT P.I.s.
THEY'VE BEEN HELPFUL BUT IT'S
STILL A WORK IN PROGRESS.
I HOPEFUL CONTINUE TO SHAPE THE
MEMBERSHIP OF EACH OF THESE
AFFINITY GROUPS.
IN TERMS OF BUDGET AND PERSONNEL
ISSUES, THE DIVISION OF
INTRAMURAL RESEARCH INCORPORATED
ABOUT 14% OF THE OVER ALL
ALLOCATION TO HE NICHD.
-- THE NICHD.
OF THAT WE'RE ABOUT $215 MILLION
AND APPROXIMATELY 40% OF THAT
GOES TO LAB CONSUMABLES AND
SALARIES AND ABOUT 40% TO
CLINICAL CENTER ASSESSMENT AND
SUPPLY FUNDS AND THIS GOES TO
OVERHEAD AND DON'T HAVE CONTROL
HOW THAT IS USED AND IT'S USED
ON CLINICAL CENTERS AND
INITIATIVES AND KEEPING THE
LIGHTS ON AND SO FORTH.
OF THAT BUDGET, ONLY ABOUT 55%
OF THAT IS FLUID AND SALARIES
ARE A NEVER INCREASING ENTITY
AND THE ONLY PLACE WE'RE ABLE TO
GET RESPITE FROM THAT IS TO BITE
INTO THE BUDGETS FOR RESEARCH
LABS.
WE'RE CONSTANTLY AWARE OF
RE-EVALUATING HOW LABS ARE
FUNDED AND IT'S A HUGE
INITIATIVE IN EVERY ORGANIZATION
TO MAKE SURE THE MONEY AVAILABLE
FOR RESEARCH IS NOT COMPLETELY
OVERWHELMED BY THE
EVER-INCREASING SALARY COSTS.
WE HAVE A SMALL POOL OF MONEY
WHICH WE KEEP FOR RENOVATIONS AS
WELL AS EQUIPMENT AND THEY TEND
TO FLUCTUATE AND WE CAN BUY $1
MILLION AND $3 MILLION IN
CAPITAL EQUIPMENT AS THE YEAR
PROGRESSES.
THOSE NUMBERS DIDN'T INCLUDE THE
DIFFERENT BUDGET AND ABOUT $20
MILLION INCLUDED IN R&D FUNDING
WHICH IS FOR CONTRACEPTIVE
DEVELOPMENT AND OTHER R&D
BUDGETS EMBEDDED IN SITE.
WE HAVE HALF A NUMBER OF
POST-DOCS UPWARDS OF 4-6 YEARS.
WE HAVE CLOSE TO 100 POST-BACS
AND WE HAVE GRADUATE STUDENTS
AND THERE'S MORE INCOMING AND 14
CLINICAL FELLOWS.
THIS YEAR WE HAD OUR FIRST
IN-PERSON SUMMER INTERNSHIP
PROGRAM IN YEARS.
IT WAS A HYBRID MODEL WITH SOME
THAT DIDN'T NEED TO BE IN LABS
WORKING VIRTUALLY BUT THE VAST
MAJORITY WERE BACK IN PERSON IN
LABS.
THAT WAS INCREDIBLY SUCCESSFUL
TO HAVE PEOPLE BACK ON CAMPUS
AGAIN DOING SUMMER INTERNSHIP
AND HAD ABOUT 50 WHICH IS A
USUAL AVERAGE NUMBER FOR OUR
SUMMER INTERN PROGRAM SO I KIND
OF FEEL LIKE WE'RE ALMOST BACK
TO FULL STRENGTH ON OUR
INTERNSHIP PROGRAM.
THERE'LL BE A NUMBER OF STAFF
CHANGES.
WE HAD AN ACTING DIRECTOR AND
WAS PREVIOUSLY THE DEPUTY
DIRECTOR AND SHE'S A PERINATAL
EPIDEMIOLOGIST FOCUSSING ON
FETAL GROWTH AND PREGNANCY
OUTCOMES WITH A RELATION TO
MATERNAL INTEREST.
AND WE HAVE AN ACTING CLINICAL
DIRECTOR.
SO DANNY PORTER OUR PREVIOUS
CLINICAL DIRECTOR STEPPED DOWN
FROM A ROLE IN MARCH OF THIS
YEAR.
HE HAD SERVED IN THAT ROLE MORE
THAN 11 YEARS.
I'M HAPPY TO SAY LA VERNE WHO
HAD BEEN SERVING SINCE 2020
GRACIOUSLY AGREED TO SERVE AS
THE NEW ACTING CLINIC DIRECTOR
UNTIL WE APPOINT A NEW DIRECTOR
AND A SEARCH IS CURRENTLY
UNDERWAY FOR A PERMANENT
CLINICAL DIRECTOR.
DR. MENSAH HAS UNIQUE
OPERATIONAL AND CLINICAL
RESEARCH EXPERIENCE.
WE'VE HAD A COUPLE ASSOCIATE
SCIENTIFIC DIRECTOR POSITIONS I
WANT TO SHARE WITH DANNY
STEPPING DOWN AND I WAS KEEN TO
USE EXPERTISE AND BRIDGE ROLES
BETWEEN BASIC AND CLINICAL
PROGRAMS AND WAS GLAD WHEN HE
WAS MADE THE ASD FOR CLINICAL
RESEARCH.
WE APPOINTED STEPHEN GILMAN AS
OUR EQUITY, DIVERSITY, INCLUSION
AND ACCESSIBILITY PERSON.
AND PART OF STEPHEN'S ROLE WAS
TO HELP DOVETAIL BETWEEN THE
REAP AND STRIVE INITIATIVES AND
HELP IN THE UNITE INITIATIVES.
HE'S TAKEN ON THE ROLE.
TWO NEW SCIENTIFIC DIRECTOR
POSITIONS WE'VE HAD TURN OVER IN
OUR RECRUITMENT AND RETENTION
AND WE HAVE A NEW ASD AND DEPUTY
ASD AND I ASKED THEM TO PUT
THEIR EMPHASIS ON RETENTION PART
AND FOCUS ON WAYS TO CHANGE OUR
CAREER DEVELOPMENT INSIDE ASD
AND TO LOOK TO TENURED AND
RECENTLY TENURED AND MID-CAREER
SCIENTISTS TO COME UP WITH
INITIATIVES TO INTRODUCE TO
CAREER COACHING AND THINKING
ABOUT REN MORING ROLES AND
TRYING TO COP UP WITH WAYS TO
ENGAGE THEM AS THEY AND I HAVE
STAFF SCIENTISTS AND THEY'RE
POPULATION IS TYPICALLY
OVERLOOKED IN THE CONVERSATIONS
AS FAR AS CAREER DEVELOPMENT AND
MENTORING.
AND IN FORMING THE NEW ASD
PARTNERSHIP WE STARTED A
COMMITTEE OF STAFF SCIENTIST TO
FIND OUT WHAT IT IS THEY FEEL
THEY'RE MISSING OR NEED.
SOME INITIATIVES IS TO HAVE
RETRAINING OPPORTUNITIES OR
LEARNING OF NEW TECHNIQUES
ACROSS LABS AND I.C.s AND
PROVIDING BETTER MENTORING AND
LEARNING SKILLS AND ESTABLISHING
A NEW AWARD SYSTEM SPECIFICALLY
FOR STAFF SCIENTISTS TO ALLOW
THEM TO TRAVEL TO MEETING AS
THEY WOULDN'T OTHER WISE BE
ALLOWED TO AND THIS IS FINALLY
GETTING SOME ATTENTION.
WE ALSO ESTABLISHED AN NICHD
AWARDS COMMITTEE.
THIS STEMMED FROM A CONVERSATION
I HAD WITH OTHER SCIENTIFIC
DIRECTORS.
WE DON'T DO A PARTICULARLY GREAT
JOB AT SHOWCASING AND
HIGHLIGHTING THE SCIENCE MANY OF
OUR INCREDIBLY TALENTED
SCIENTISTS DO AND PUT THEM
FORWARD FOR AWARDS AND WE WANTED
TO ENGAGE OUR P.I.s TO PROVIDE A
DATABASE OF POSSIBLE AWARDS OF
NOT ONLY STUDENTS AND POST-DOCS
BUT P.I.s WOULD BE ELIGIBLE FOR.
WE'VE ALREADY SEEN THE FRUITS OF
THE LABOR.
THIS HAS BEEN GOING THREE OR
FOUR MONTHS AND THIS COMMITTEE
RECENTLY LED THE NOMINATION AND
IT WAS JUST AWARDED THE AMERICAN
SOCIETY AND IT'S VERY ENGAGED
AND I HAVE BEEN BOWLED OVER BY
THE ENTHUSIASM THE INITIATIVES
HAVE BEEN MET WITH.
WE ALSO HAD STAFF CHANGES IN OUR
ANIMAL PROGRAM AND OUR ANIMAL
PROGRAM DIRECTOR AND SENIOR VET
STEPPED DOWN TO TAKE ON A CAREER
ELSEWHERE.
THAT WAS A HUGE LOSS FOR US.
FORTUNATELY, RACHEL LABITT WHO
WAS TRAINING IN HER DEPUTY HAS
STEPPED UP AND APPOINTED AS THE
SENIOR VETERINARIAN UNTIL WE
FIND A PERMANENT REPLACEMENT.
WE HAVE A BURGEONING NUMBER OF
INITIATIVES THE OFFICE UNDER
TAKES.
THIS SHOWS A BUNCH FLOW DIAGRAMS
OF THE TYPES OF PROGRAMS AND
INITIATIVE THE OFFICE OF
EDUCATION PROVIDE FROM
LEADERSHIP AND MANAGEMENT
TRAINING APPLYING FOR AND THE
INTRODUCING IDEAS OF RESEARCH
AWARDS, OFFERING TEACHING, NOT
ONLY INSIDE THE INTRAMURAL
PROGRAM BUT AT LOCAL
UNIVERSITIES.
CAREERS, RESEARCH ETHICS
TRAINING, COMMUNICATION, WE HAVE
A RETREAT AND PREPARING FOR
MEDICAL AND GRADUATE SCHOOL
APPLICATION AND GRANTS AND
WRITING.
THIS IS SPEARHEADED BY A FORMER
POST-DOC INSIDE THE INTRAMURAL
PROGRAM WHO BECAME THE DEPUTY OF
THE OFFICE OF EDUCATION AND
STEPPED UP TO BECOME THE
DIRECTOR.
SHE HAS AN INCREDIBLE ENERGY AND
HER PORTFOLIO IS EVER INCREASE
AND HAS DONE THINGS LIKE
PRACTICING FOR PUBLIC SPEAKING
AND TEACHING AND INCLUDING
THINGS LIKE JOB TALKS OR
INDIVIDUAL COACHING,
THREE-MINUTE ELEVATOR SPEECHES,
DEVELOPMENT ACROSS UNIVERSITIES
AND JUST TRYING TO ENGAGE ALL
TRAINEES AT ALL LEVELS IN BEING
ABLE TO SPEAK PUBLICLY AND TALK
DURING THEIR MONTHLY P.I.
MEETINGS.
WE HAVE MANY MANY AND WE HAVE
FUNDING AND PRACTICE FOR JOB
INTERVIEWS PREPARATION AND HOW
TO NETWORK AND INDIVIDUALIZED
GUIDANCE AND SO FORTH.
IT'S INCREDIBLY LABOR INTENSIVE
AND ERIN AND HER STAFF HAVE
TAKEN TO THESE.
AND WE'VE TRIED TO TRANSITION
BACK TO REASONABLE OCCUPANCY IN
LABS ON CAMPUS.
IT BECAME CLEAR AS ALL ARE AWARE
WE ALREADY HAD A SHORT BRIEF
CONVERSATION ABOUT MENTAL HEALTH
ISSUES ACROSS OUR TRAINEES BUT
IT BECAME CLEAR THAT A LOT OF
OUR YOUNG TRAINEES IN PARTICULAR
POST-DOCS AND POST-BACS HAVE
SUFFERED FROM BEING IN ISOLATION
AND HAVING LIMITED INTERACTION
FROM HER LAB AND I ASKED ERIN
AND HER DEPPY -- DEPUTY TO MEET
WITH POST-BACS AND SO ON AND
UPWARDS OF 300 INDIVIDUALS AND I
NEVER OCCURRED TO ME HOW LONG
AND HOW MANY TIMES MEETING WITH
300 PEOPLE AND WHAT A HERCULEAN
EFFORT IT WILL TAKE TO PULL THAT
OFF.
OVER THE COURSE OF MONTHS, ERIN
AND TERESA MET WITH EVERY SINGLE
INDIVIDUAL TRAINEE INSIDE OUR
I.C.
IT WAS A VERY A VERY INFORMATIVE
AND ILLUMINATING EXPERIENCE AND
WE COULD MAKE EDUCATED GUESTS
ABOUT THE ISSUES OUR TRAINEES
WERE SUFFERING FROM BUT MANY
THINGS WE LEARNED AND WERE TOLD
AND WERE TAKEN BY SURPRISE BY.
THIS WAS A MAJOR UNDERTAKING AND
WE'RE ON OUR SECOND YEAR OF
GOING THROUGH THE EXERCISE AND I
KNOW ERIN STARTED SETTING UP FOR
INTERVIEWING TRAINEES.
THIS IS THE LEVEL OF INTERACTION
WITH TRAINEES.
WE DO A LARGE NUMBER OF WEBINARS
WITH INDUSTRY JOBS AND OTHER
OUTSIDE REGULAR AND P.I. OR LAB
CHIEF AND SCIENTIFIC CAREERS.
WE HAVE ANNUAL GRANT WRITING
WORKSHOPS.
WE INITIATED WITH OTHER I.C.s A
THREE-MINUTE TALK A COMPETITIVE
COMPETITION THAT STARTED OFF
WITH THREE INSTITUTES AND NOW
EXPANDED TO 12 INSTITUTES.
IT'S AN INCREDIBLY FUN
UNDERTAKING.
EACH TRAINEE GETS A NUMBER OF
HOURS WITH A SPEAKING COACH AND
PRESENTATION DOWN TO THREE
MINUTES AND ONE SLIDE AND
COMPETE AGAINST EACH OTHER AND
IT STARTED WITH THREE
INSTITUTES.
WE NOW HAVE 12.
IT'S AN INCREDIBLY FUN
EXPERIENCE.
WE'RE JUST ABOUT TO HAVE OUR NIH
FELLOWS AWARD AND IT WENT TO
FOUR FELLOWS.
IT'S IN ITS FIFTH CYCLE AND
WE'RE CURRENTLY DEVELOPING AN
ANNUAL PROGRESS REPORT SYSTEM
THAT INCLUDES OUR POSTBAC
FELLOWSHIP AND WE HAVEN'T DONE
SOMETHING SIMILAR FOR OTHERS AND
WE'RE ABOUT TO CHANGE THAT AND
WE'RE ALWAYS CURIOUS TO SPEAK TO
TRAINEES AS THEY LEAVE THE NIH.
WE WANT TO HEAR ABOUT THEIR
EXPERIENCES GOOD AND BAD AND
CONSIDERING AN EXIT SURVEY.
THE LAST THING I WANT TO SPEND
TIME ON IS TALKING ABOUT OUR
HOWARD UNIVERSITY PARTNERSHIP.
THIS WAS AN AGREEMENT ENTERED
WITH HOWARD UNIVERSITY BY THE
NIH AT LARGE IN THE CLINICAL
CENTER BACK IN 2019 AND THE
INTENT WAS TO FACILITATE A MODEL
NETWORK FOR RESEARCH
COLLABORATIONS AND TRAINING.
WE HAVE OTHER INSTITUTES THAT WE
ARE ALSO INVOLVED WITH AT THE
UNIVERSITY OF MARYLAND AND
VIRGINIA COMMONWEALTH
UNIVERSITY.
WE'RE INVOLVED WITH FOUR MAJOR
INITIATIVES WITH HOWARD.
ONE IS THE COLLEGE OF ARTS AND
SCIENCES THE BIOLOGY HONOR
PROGRAM WHERE WE PROVIDE
SECONDARY RESEARCH MENTORSHIP.
WE HAVE P.I.s THAT PROVIDE
STUDENTS THE OPPORTUNITY FOR A
TWO-YEAR PERIOD OF ADVANCED
MENTORING INSTRUCTION.
AND COORDINATED WITH OUR SUMMER
INTERNSHIP PROGRAM SO THERE'S
CONTINUITY THROUGH THEIR
ACADEMIC YEAR AND HAD FOUR
STUDENTS STARTED LAST YEAR AND
THEY'RE GOING TO CONTINUE INTO
THEIR SECOND YEAR AND HAVE A NEW
CYCLE OF SIX STUDENTS NOW PAIRED
WITH MEMBERS IN THE DIR.
AND MENTORS TO THREE AND FOUR
MEDICAL STUDENTS AND HAVE ONE
PAIRING CONFIRMED FOR LAST YEAR.
WE HAVE SEVERAL FOR P.I.s GIVING
LECTURES AND PRESENT TO THE
STUDENTS THERE AND RAN INTO THE
NINDS OFFICE OF EDUCATION AND
WAS OUR DIRECTOR BEFORE SHE
MOVED TO NINDS.
YVETTE AND OTHERS STARTED A
SIX-WEEK SEMINAR SERIES LAUNCHED
OVER THE COURSE OF THE LAST
YEAR.
AND IT GIVES EXPOSURE TO MEDICAL
AND RESEARCH CAREERS AND PROVIDE
GUIDANCE HOW TO PROCEED WITH THE
ADMISSIONS PROCESS.
ABOUT A MONTH AGO STEVEN GILMAN
AND MYSELF MET WITH THE DOZEN OR
SO STUDENTS THAT HAD TAKEN THIS
COURSE FOR THE SECOND TIME.
WE RAN IT FOR THE FIRST TIME A
YEAR AGO IN JULY AND RAN IT
AGAIN THIS YEAR AND THIS TIME WE
HAD 12 STUDENTS AND THEY WERE
GIVEN THE OPPORTUNITY TO HAVE A
FIVE-MINUTE PRESENTATION AND ONE
SLIDE AND THE LEVEL OF
ENTHUSIASM FROM THE STUDENTS WAS
OVERWHELMING.
IT WAS ONE OF THE MOST FUN
EXPERIENCES I CAN SAY I'VE HAD
AS ACTING SCIENTIFIC DIRECTOR.
THEY'RE ENTHUSIASM WAS LEAPING
OFF THE SCREEN.
AND WHAT WAS INFORMATIVE IS ALL
OF THEM HAD PRETTY MUCH COME
INTO THE PROGRAM THINKING THEY
KNEW WHAT THEY'RE CAREER PLAN OR
CAREER PATH WAS GOING TO BE.
AND BY THE TIME THEY LEFT, THEY
HAD COMPLETELY DONE AN ABOUT
TURN AND THOUGHT ABOUT FIELDS OF
RESEARCH AND CAREERS THEY HAD
NEVER REALLY EVEN THOUGHT ABOUT
OR BEEN EXPOSED TO.
IT WAS AN INCREDIBLY GRATIFYING
OPPORTUNITY TO LISTEN TO A DOZEN
OR SO STUDENTS ABOUT TO EMBARK
ON THEIR CAREERS IT WAS A REAL
SUCCESS FROM THE OFFICE OF
EDUCATION.
AND WITH THAT I'LL LEAVE IT AND
HAPPY TO ANSWER ANY QUESTIONS
ANY OF YOU MAY HAVE.
>> IT'S GREAT TO HEAR HOW THINGS
ARE GOING IN THE INTRAMURAL
PROGRAM AND CONGRATULATIONS.
I LIKE THE LAST PROGRAM YOU AND
THOUGHT IT WAS SPECTACULAR TO DO
IT WITH THE OB-GYN INTEREST
GROUP.
WHAT ABOUT PEDIATRIC INTEREST
GROUP?
IS THAT A POSSIBILITY?
NOW THAT YOU HAVE ONE AREA CAN
YOU ADD ANOTHER AREA?
>> THE NUMBER OF PERSONNEL WE
HAVE THAT ARE QUITE SMALL AND A
NARROW FOCUS.
WE PUT OUR ENERGY INTO THESE
FOUR WHICH HAVE BEEN SUCCESSFUL.
WE DON'T HAVE A DIRECTOR PLAN
BUT WE'RE INTERESTED IN
EXPLORING OTHER DIRECTIONS FOR
THAT INITIATIVE.
>> GREAT WAY TO DO THIS, CHRIS.
THANKS.
>> THANK YOU.
CHRIS.
CHRIS WILL BE SPEAKING AGAIN
TOMORROW IN THE CLOSED SESSION.
SO YOU'LL HAVE ANOTHER CHANCE TO
ASK QUESTIONS OF HIM WHILE YOU
DIGEST THE INFORMATION THAT YOU
HEARD SO FAR.
THANK YOU VERY MUCH FOR THAT
VERY INFORMATIVE OVERVIEW.
AND NOW WE ARE GOING TO HEAR A
PRESENTATION FROM DR. CRISWELL
ONE OF THE COVID FIVE.
SO WE CALL THEM THE COVID FIVE
DIRECTORS BECAUSE OVER THE
PERIOD OF THE PANDEMIC, FIVE NEW
INSTITUTE DIRECTORS WERE
APPOINTED AND SO THEY FORM A
COHORT AMONGST THEMSELVES AND
THEY BRAVELY INITIATED THEIR NEW
ROLES WHILE THERE WERE SO MANY
THINGS GOING ON IN THE PANDEMIC.
BUT IT'S A GREAT PLEASURE TO
INTRODUCE MY COLLEAGUE,
DR. CRISWELL WHO WAS APPOINTED
AS THE DIRECTOR OF THE NATIONAL
INSTITUTE OF ARTHRITIS AND
MUSCULOSKELETAL AND SKIN DISEASE
KNOWN AS NIAMS IN FEBRUARY OF
2021.
SO PRIOR TO JOINING,
DR. CRISWELL WAS THE VICE
CHANCELLOR OF RESEARCH AT THE
UNIVERSITY OF CALIFORNIA SAN
FRANCISCO.
SHE'S A BOARD CERTIFIED
RHEUMATOLOGIST.
SHE WAS ALSO A PROFESSOR OF
RHEUMATOLOGY AND PROFESSOR OF
ORAL FACIAL SCIENCES AT UCSF.
SHE WAS AWARDED THE KENNETH H.
FREI ENDOWED CHAIR IN
RHEUMATOLOGY AND DISTINGUISHED
PROFESSORSHIP IN RHEUMATOLOGY.
HER OWN LABORATORY'S RESEARCH
INTERESTS ARE ON THE GENETICS
AND EPIDEMIOLOGY OF HUMAN
AUTOIMMUNE DISEASE PARTICULARLY
RHEUMATOID ARTHRITIS.
SHE'LL BE TELLING US ABOUT THE
MISSION TO SUPPORT RESEARCH IN
THE CAUSES, TREATMENT AND
PREVENTION OF ARTHRITIS IN
MUSCULOSKELETAL SKIN DISEASES
AND THE NEWEST STRATEGIC
PLANNING EFFORT SHE IS
LAUNCHING.
AND AS WE HAD ASKED OF THE OTHER
NEW DIRECTORS WHEN THEY COME TO
SPEAK WITH US, WE'RE
PARTICULARLY INTERESTED IN AREAS
IN WHICH OUR TWO INSTITUTES MAY
COLLABORATE.
DR. CRISWELL, OVER TO YOU.
>>
>> THANK YOU.
I APPRECIATE THE OPPORTUNITY TO
SPEAK WITH YOU AND TO MEET YOU
ALBEIT IN THIS VIRTUAL FORMAT.
THANK YOU FOR THE OPPORTUNITY.
IT'S A PLEASURE TO BE HERE AND
APPRECIATE THE OPPORTUNITY TO
SHARE HIGHLIGHTS OF THE
LONG-TERM COMMITMENT TO RESEARCH
AREAS WHERE THE MISSIONS OF
INSTITUTES INTERSECT.
THE TWO ENJOYED A PARTNERSHIP
AND LOOK FORWARD TO A ROBUST
DISCUSSION.
AS THE DIRECTOR I'M EXCITED TO
EVERY SEE THE WIDE RANGE
PORTFOLIO AND I HAVE BEEN
GETTING TO GET TO KNOW MY FELLOW
DIRECTORS AND EXPLORING WAYS TO
PARTNER IN MANY AREAS OF COMMON
INTERESTED.
I APPRECIATED DR. BIANCHI'S
SUPPORT AND ADVISE DURING MY
RECRUITMENT AND DURING THE
ONBOARDING PROCESS.
THE INSTITUTE WAS ESTABLISHED IN
1986 BUT WAS PART OF THE
NATIONAL INSTITUTE OF ARTHRITIS,
DIABETES AND DIGESTIVE AND
KIDNEY DISEASES NOW THE NATIONAL
INSTITUTE OF DIABETES AND
DIGESTIVE AND KIDNEY DISEASE.
AND LOOK INTO CAUSES, PREVENTION
TREATMENT OF ARTHRITIS AND
MUSCULOSKELETAL AND SKIN
DISEASES AND PROVIDE INFORMATION
TO THE PUBLIC OF OUR RESEARCH
PROGRESS IN MISSION AREAS AND
HOW IT CAN BE APPLIED TO IMPROVE
HEALTH.
IN FUND YEAR 2022 OUR MISSION
WAS CARRIED OUT BY $656 MILLION
AND NIAMS SUPPORTS THE
INTRAMURAL RESEARCH PROGRAM AND
OUR FEDERAL LABORATORIES AND
EXTRAMURAL RESEARCH BY
UNIVERSITIES AND INSTITUTIONS
ACROSS THE COUNTRY.
THIS SHOWS OUR FUNDING AMONG THE
INTRAMURAL, EXTRAMURAL AND OTHER
COMPONENTS OF THE NIH AS WELL AS
NIAMS.
NIAMS INVESTS MOST MONEY AT
INSTITUTIONS AROUND THE COUNTRY
AND SPEND ON THE PROGRAM HERE IN
BETHESDA.
WITH INPUT FROM THE NIAMS
ADVISORY COUNCIL AND DEVELOPED
THE PLAN COVERING FISCAL YEARS
20 TO 24 TO IMPROVE HEALTH
OUTCOMES IN THE FIVE CORE AREAS
OF THE NIAMS MISSION LISTED ON
THE SLIDE AND INCLUDE THE
SYSTEMIC AND RHEUMATIC DISEASES
AND BONE BIOLOGY AND DISEASES
AND MUSCLE BIOLOGY AND DISEASES
AND JOINT BIOLOGY AND DISEASES
AND PEDIATRICS RESEARCH.
THERE'S DISEASES AFFECTING
PEDIATRIC POPULATIONS ACROSS THE
NIAMS MISSION AREAS.
ARTHRITIS AND MUSCULOSKELETAL
AND SKIN DISEASES AFFECT ALL
POPULATIONS AND CAUSE TREMENDOUS
HUMAN SUFFERING AND AFFECT THE
U.S. ECONOMY DUE TO HEALTH CARE
COST AND LOSS PRODUCTIVITY.
MANY WHETHER COMMON OR RARE ALSO
AFFECT WOMEN DISPROPORTIONATELY
AND SOME WITH INCREASED DISEASE
SEVERITY DURING PREGNANCY.
THE CURRENT NIAMS STRATEGIC PLAN
ALSO HIGHLIGHTS SEVERAL
SCIENTIFIC THEMES THAT COVER
ACROSS OUR MISSION AREAS.
THESE INCLUDE USING NEW
TECHNOLOGIES TO IDENTIFY SHARED
MECHANISMS IN HEALTH AND AMONG
DISEASES, ADVANCING
PATIENT-CENTERED APPROACHES,
ENABLING PRECISION MEDICINE TO
TAILOR CARE FOR EACH PATIENT,
AND ADDRESSING THE HEALTH NEEDS
OF DIVERSE POPULATIONS.
THESE TOPICS EMERGE FROM NIAMS
LISTENING SESSIONS WITH THOUGHT
LEADERS IN EACH OF OUR DISEASE
AND TISSUE SPECIFIC MISSION
AREAS.
AS A FIRST STEP WE PUBLISHED A
REQUEST FOR INFORMATION TO
SOLICIT PUBLIC INPUT ON OLD
ASPIRATIONS AS WELL AS
CROSS-CUTTING RESEARCH
OPPORTUNITIES MANY OF WHICH
RELATE TO SCIENTIFIC AREAS THAT
ARE ALSO OF INTEREST TO THE
NICHD I'D LIKE TO INVITE ALL TO
PARTICIPATE AND INVITE FOR INPUT
AS WE PUT FORWARD OUR NEXT STUCK
PLAN.
BEYOND OUR STRONG PLANNING
EFFORT COMMUNITY ENGAGEMENT AND
PATIENT VOICE HAS BEEN CRITICAL.
AND ACTS WITH AN INDEPENDENT
CONSORTIUM OF PROFESSIONAL AND
VOLUNTARY ORGANIZATIONS SHARING
INTEREST IN THE NIAMS MISSION
AREAS.
NIAMS IS EAGER TO LEVERAGE THIS
PARTNERSHIP TO ADVANCE PEDIATRIC
RESEARCH IN OUR PORTFOLIO AND
SAY LONG-TERM SUPPORTER OF THE
PEDIATRIC DERMATOLOGY RESEARCH
ALLIANCE AND IT'S ANNUAL MEETING
THROUGH THE CONFERENCE GRANT
WITH THE R13 MECHANISM.
PREVIOUSLY, NIAMS SUPPORTED THE
ARTHROSCLEROSIS STUDY KNOWN AS
APPLE WHICH WAS A COLLABORATIVE
EFFORT WITH THE CHILD ARTHRITIS
AND RESEARCH A.
LUPUS IN PEDIATRIC POPULATIONS
IS GENERALLY MORE SEVERE THAN IN
ADULTS WITH COMORBIDITIES AND
THEY LOOKED AT TREATMENT TO SLOW
PROGRESSION OF ATHEROSCLEROSIS
IN CHILDREN WITH LUPUS.
WHILE THE RESULTS DO NOT
DEMONSTRATE COMPELLING EVIDENCE
FOR TREATMENT WITH STATINS FOR
THE PREVENTION OF
ATHEROSCLEROSIS THERE MAY BE
BENEFITS TO PATIENT SUB GROUPS
WITH SEVERE DISEASE AND SUCH
STUDIES ARE ONGOING.
WITHIN THE NIH, IN ORDER TO
LEVERAGE OPPORTUNITIES FOR
PARTNERSHIP AND EVIDENCE OF
NIAMS COMMITMENT TO PEDIATRIC
RESEARCH THE INSTITUTE IS AN
ACTIVE MEMBER OF THE NIH
PEDIATRIC CONSORTIUM LED BY
NICHD.
BEFORE DISCUSSING CURRENT
ACTIVITIES I'D LIKE TO NOTE THE
SUCCESSFUL PAST COLLABORATION.
IN 2012, NIAMS PARTICIPATED IN
THE LAUNCH OF THE TRANS-NIH
ENVIRONMENTAL INFLUENCES ON
CHILD HEALTH OUTCOMES ALONG WITH
NICHD AND OTHER I.C.s.
THIS PART OF ECHO, NIAMS OVERSAW
THE VALIDATION OF PEDIATRIC
PATIENT-REPORTED OUTCOMES IN
CHRONIC DISEASE CONSORTIUM
COMMONLY REFERRED TO AS PEPPER
ON BEHALF OF THE NIH.
THE GOAL OF THE PEPPER
CONSORTIUM WAS TO TEST SEVERAL
PEDIATRIC PATIENT-REPORTED
OUTCOME TOOLS THAT MEASURE
ASPECTS OF PHYSICAL, MENTAL AND
SOCIAL WELL BEING SUCH AS PAIN,
ANXIETY AND PEER RELATIONSHIPS.
IT USED PEDIATRIC SELF-REPORT
AND PARENT PROXY INSTRUMENTS
INVOLVED THROUGH THE NIH COMMON
FUND'S PROMISE OF
PATIENT-REPORTED OUTCOMES AND
SYSTEM INITIATIVE.
THE CONSORTIUM FACILITATED THEIR
ADOPTION AND MEANINGFUL USE IN
CLINICAL CARE SETTINGS.
NEXT, I WANT TO COVER CURRENT
EXAMPLES OF PEDIATRIC RESEARCH
AT NIAMS STARTING WITH OUR
INTRAMURAL RESEARCH PROGRAM
WHICH HAS A STRONG FOCUS ON
PEDIATRIC DISEASE RESEARCH
PARTICULARLY FOR RHEUMATIC
DISEASES.
OUR CLINICAL DIRECTOR,
DR. ROBERT CULVERT IS A
PEDIATRIC RHEUMATOLOGIST AND HIS
RESEARCH FOCUSES ON
UNDERSTANDING THE ETIOLOGY AND
PATHOGENESIS OF EARLY ONSET OF
JUVENILE SPONDAL ARTHRITIS AND
DR. KIM FOCUSES ON EXPLORING THE
PATHOGENESIS AND INVOLVE
TREATMENTS IN A CHRONIC
AUTOIMMUNE INFLAMMATORY DISEASE
RESULTING IN MUSCLE WEAKNESS AND
SKIN RASHES WITH THE POTENTIAL
FOR INVOLVEMENT OF OTHER ORGANS.
DR. OMBRELLO CERTIFIED IN ADULT
AND PEDIATRIC RHEUMATOLOGY LEADS
OUR TRANSLATIONAL GENETICS AND
GENOMIC UNIT.
HE FOCUSES ON UNDERSTANDING THE
MECHANISM UNDER LIE INFLAMMATORY
AUTOIMMUNE DISEASE WITH COMPLEX
DISEASES SUCH AS STIHL'S DISEASE
AND OTHERS.
THE LUPUS GENOMICS AND GLOBAL
HEALTH DISPARITIES UNIT LED BY
DR. LEWANDOWSKI INCLUDES DIVERSE
POPULATIONS OF LUPUS PATIENTS
AND THEIR STUDIES OF GENETIC
CHANGES THAT OCCUR IN EARLY
ONSET LUPUS.
AND WE ALSO RECENTLY RECRUIT A
PEDIATRIC DERMATOLOGIST TO JOIN
OUR CUTANEOUS MICROBIOME
SECTION.
IT KOVRZ OF COVERS
>> COVERS LOOKING AT DERMATITIS
AND OTHERS.
AND OTHER INVESTIGATORS ALSO
COLLABORATE WITH THEIR
INTRAMURAL COLLEAGUES.
ONE EXAMPLE IS A STUDY OF A RARE
PROGRESSIVE DISEASE THAT AFFECTS
THE GROWTH AND DEVELOPMENT OF
BONES AND SOFT TISSUE.
THE NIAMS CLINICAL UNIT AND
COLLEAGUES WORKED WITH 15
PATIENTS FROM AROUND THE WORLD
WHO TRAVELED TO THE NIH CLINICAL
CENTER TO UNDER GO BIOPSIES OF
AFACT AND UNAFFECTED BONES AND
MANY HAD MAP2K1 A GENE CRITICAL
FOR SKELETAL DEVELOPMENT.
THE MUTATION COVERED BONE CELLS
TO DISPLAY INCREASED GROWTH AND
HARDENING IN THE AFFECTED BONES.
IN ADDITION TO PROVIDING NEW
INSIGHT INTO THIS CONDITION,
THEY'RE FINDING IMPLICATIONS FOR
OTHER DISEASES THAT IMPACT
BROADER POPULATIONS SUCH AS THE
AFFECTS OF AGING ON BONE.
NEXT, I'LL PROVIDE UPDATES OF
EXTRAMURAL RESEARCH ACROSS THE
MISSION AREAS RELATED TO HUMAN
DEVELOPMENT AND CHILD HEALTH
STARTING WITH AN EXAMPLE FROM
OUR SYSTEMIC RHEUMATIC DISEASES
PORTFOLIO.
AS YOU MAY KNOW AN ESTIMATED 90%
OF PEOPLE DIAGNOSED WITH LUPUS
IS WOMEN AND MORE PREVALENT IN
AFRICAN AMERICAN AND HISPANICS
AND ASIANS THAN WHITES.
WOMEN HAVE BEEN COUNSELLED TO
AVOID WITH PREGNANCY AS
PREGNANCY THROMBOSIS IS FAIRLY
COMMON IN THESE PATIENTS
PARTICULARLY THOSE WHO HAVE
CERTAIN ANTIBODIES.
NIAMS SUPPORTED THE PROMISE
STUDY WHICH STANDS FOR
PREDICTORS OF PREGNANCY
OUTCOMES, BIOMARKERS IN ANTIFOSO
LIPID ANTIBIOTIC SYSTEM LED BY
THE HOSPITAL BY SPECIAL SURGERY
AND THERE WERE OVER 700 PATIENTS
WITH LUPUS OR THE PHOSSO LIPID
ANTIBIOTIC SYSTEMS.
AND PAPERS PUBLISHED WITH NYU
DEMONSTRATED MOST WOMEN WHO HAVE
LUPUS CAN EXPECT A GOOD
PREGNANCY OUTCOME IF THEIR
DISEASE IS INACTIVE.
BUT INVESTIGATORS ALSO IDENTIFY
MOLECULE IN SOME WOMEN'S BLOOD
THAT INDICATE THE POSSIBILITY
FOR MORE SERIOUS COMPLICATIONS.
THOSE AT RISK OF MISCHARGE OR
OTHER SERIOUS COMPLICATIONS WILL
ALLOW TO TAILOR ASSESSMENT AND
COUNSELLING AND TREATMENT FOR
WOMEN WITH LUPUS.
AND THERE'S AND LOOK AT
PREVENTIBLE MATERNAL MORTALITY
AND DECREASE MATERNAL MORBIDITY
AND PROMOTE MATERNAL HEALTH
EQUITY.
WE LOOK FORWARD TO PARTNERING
WITH NICHD ON THE IMPORTANT AND
TIMELY EFFORT.
THE MUSCULAR DYSTROPHIES ARE
ANOTHER AREA OF SHARED INTEREST
OF NIAMS AND NICHD.
BOTH INSTITUTES ARE CHARTER
MEMBERS OF THE COORDINATING
COMMITTEE.
A FLAGSHIP OF THE NIH RESEARCH
PORTFOLIO IS FROM THE
MUSCULODYSTROPHY RESEARCH
CENTERS A COLLABORATION AMONG
NIAMS, NICHD AND NINDS AND
NHLBI.
THE GOAL OF THE PROGRAM IS TO
HAVE SIX ACTIVE CENTERS AT ANY
ONE TIME WITH NIAMS, NICHD AND
SUPPORTING TWO CENTERS EACH AND
NHLBI SUPPORTING CENTERS THAT
RELATE TO CARDIAC OR PULMONARY
CENTERS.
ONE IS AT THE UNIVERSITY OF
FLORIDA WHERE INVESTIGATORS
FOCUS ON THE ROLES OF
INFLAMMATION AND FIBROSIS AND
FAT IN MUSCULODYSTROPHY AND THE
CANCER RESEARCH CENTER WHERE
INVESTIGATORS ARE CONDUCTING
CLINIC STUDIES ON DUCHENNE'S
MUSCULAR DYSTROPHY AND OTHERS.
AND THE TWO SUPPORTED BY THE
NICHD ARE AT THE UNIVERSITY OF
MASSACHUSETTS MEDICAL SCHOOL
WHERE INVESTIGATORS ARE
FOCUSSING ON MOLECULAR GENETIC
AND EPIGENETIC PATHOLOGIES AND
THE U.T. SOUTHWESTERN WHERE
THEY'RE EXPLORING DUCHENNE'S
MUSCULODYSTROPHY.
IN 2019 THE CENTER EVALUATION
WORKING GROUP WITH INTEGRAL
CONTRIBUTION FROM STAFF
INCLUDING DR. GLAVINE COMPLETED
ITS EVALUATION OF THE PROGRAM
AND PROVIDED VALUABLE
RECOMMENDATIONS TO HELP INFORM
HOW THE PROGRAM MIGHT BE
ADJUSTED TO FURTHER ADVANCE
MUSCULODYSTROPHY RESEARCH.
THE BRITTLE BONE DISORDER
CONSORTIUM ESTABLISHED IN 2014,
FOCUSES ON RARE BONE DISEASE AND
IS CO-FUNDED BY NIAMS, NCATS AND
NIDCR AND FOCUSES ON OSTEOGEN IS
IMPERFECTA WHERE BONES FRACTURE
EASILY AND WITH NO OBVIOUS CAUSE
OR INJURY.
THE CONSORTIUM HAS THREE PRIMARY
GOALS, TO GAIN A BETTER
UNDERSTANDING OF ALL GENETIC
FORMS OF OI AND EXPAND TREATMENT
OPTIONS AN IMPLEMENT TRAINING
PROGRAMS FOR THE NEXT GENERATION
OF PHYSICIANS AND SCIENTISTS IN
THE AREA OF GENETIC BONE
DISEASE.
ANOTHER PROJECT IS IMPACT.
OR INFRASTRUCTURE FOR MUSS --
THERE'S TWO FRACTURES, NEF AND
DRF IS CONTROVERSIAL AND VARIES
AMONG HEALTH CARE PROVIDERS.
IMPACT SEEKS TO DEVELOP THE
INFRASTRUCTURE AND EXPERIENCE
NECESSARY FOR MULTI-CENTER
RANDOMIZED CONTROL TRIALS.
AND THOSE WHO WANT THEIR BROKEN
BONE MOVED BACK TO ITS ORIGINAL
PLACE UNDER GENERAL ANESTHESIA
OR CONSCIOUS SEDATION WILL HAVE
HIGHER REPORTED OUTCOME SCORES
COMPARED TO THOSE WITH SIMPLE
IMMOBILIZATION ALONE AND BELIEVE
IT WILL PROVIDE FRAMEWORK AND
EXPERIENCE FOR MULTI-CENTER
CLINICAL TRIALS IN PEDIATRIC
OTHER PEDIATRICS AND WILL GUIDE
CLINIC DECISION MAKING FOR THE
DECISIONS.
LASTLY, I'D LIKE TO HIGHLIGHT
ONE ADDITIONAL AREA OF SHARED
INTEREST.
AS YOU KNOW, NICHD IS HOME TO
THE NATIONAL CENTER FOR MEDICAL
REHABILITATION RESEARCH.
AND THIS CONSISTS OF PREVENTION
AND TREATMENT STRATEGIES FOR
MITIGATING SYMPTOMS ASSOCIATED
WITH MULTIPLE CHRONIC CONDITIONS
IN INDIVIDUALS WITH PHYSICAL
IMPAIRMENTS INCLUDING PERSISTENT
PAIN.
AND THERE'S OPPORTUNITIES FOR
ENHANCED COLLABORATIONS.
NIAMS IS LEADING THE BACK PAIN
CONSORTIUM RESEARCH PROGRAM OR
BACPAC PART OF HEAL AN EFFORT TO
SPEED SCIENTIFIC SOLUTION TO
STEM THE OPIOID NATIONAL HEALTH
CRISIS.
NICHD HAS BEEN AN EARLY
SUPPORTER OF IT SINCE ITS
INCEPTION IN 2019.
CHRONIC LOW PAIN IS ONE OF THE
MOST COMMON FORMS OF CHRONIC
PAIN AMONG ADULTS AND IT RANKED
HIGHEST IN TERMS OF YEARS LIVED
WITH DISABILITY AMONG HUNDREDS
OF CONDITIONS.
CURRENT TREATMENT OPTIONS AREN'T
EFFECTIVE LEADING TO AN
INCREASED USE OF OPIOIDS.
SO BACPAC IS A TRANSLATIONAL
EFFORT TO DEVELOP PERSONALIZED
TREATMENTS FOR CHRONIC OR LOW
BACK PAIN AND WE'RE DELIGHTED TO
PLAY AN IMPORTANT ROLE IN
ADDRESSING THIS MAJOR PUBLIC
HEALTH PROGRAM AND HELP ADDRESS
THE NATION'S OPIOID EPIDEMIC.
FOR THOSE INTERESTED IN LEARNING
MORE VISIT THE NIAMS WEBSITE AND
FIND US ON FACEBOOK AND YOUTUBE.
AND TO LEARN MORE ABOUT OUR
FUNDING OPPORTUNITY
ANNOUNCEMENTS AND OUR NOTICES OF
SPECIAL INTEREST, SUBSCRIBE TO
OUR NEWSLETTER AND FOLLOW OUR
TWITTER ACCOUNTS.
AND ON THAT NOTE I WANT TO THANK
YOU AGAIN FOR THE OPPORTUNITY TO
PRESENT AND I WOULD BE HAPPY TO
TAKE ANY QUESTIONS AT THIS TIME.
I'LL STOP SHARING MY SCREEN TO
FACILITATE SOME DISCUSSION.
>> THANK YOU SO MUCH, LINDSEY.
DR. LANG, A COUNCIL MEMBER HAD
TO STEP AWAY TO TEACH A CLASS
BUT WANTED TO MENTION WE SHARE
INTEREST IN THE AREA OF MEDICAL
REHABILITATION.
YOU DID AN AMAZING JOB -- YOU
MENTIONED SOME AREAS I DIDN'T
KNOW AND INSTITUTE COLLABORATE
AND DR. OMBRELLO IS REPRESENTING
NIAMS IN THE STRATEGIC PLANNING
EFFORT FOR THE CLINICAL CENTER
ON PEDIATRIC RESEARCH.
HE'S BEEN AN ACTIVE MEMBER.
WE'VE GOTTEN TO KNOW HIM ALREADY
AND HE'S REALLY PROVIDED
IMPORTANT INSIGHT.
WITH THAT QUESTIONS.
>> I LEARNED A LOT BY PREPARING
THE PRESENTATION AND FANTASTIC
TO LEARN THE IN WAYS OUR
INSTITUTES COLLABORATED.
I HOPE NOW MOVING FORWARD WE CAN
IDENTIFY OTHER OPPORTUNITIES TO
HELP EACH OTHER AND SUPPORT OUR
SHARED INTERESTS.
>> THAT'D BE GREAT.
WAS IT SO EXCEPTIONALLY CLEAR
THERE ARE NO QUESTIONS?
>> GREAT PRESENTATION AND
EXCITING WORK.
I WANT TO REFLECT THE
INTERESTING RESULT YOU SHARED ON
THE GENETIC BURDEN OF DISEASE IN
CERTAIN PRESENTATIONS.
WE'RE SEEING THAT ACROSS
PEDIATRICS.
I THINK THERE WILL BE CLINICAL
INDICATORS WHERE RISK OF GENETIC
DISEASE IS SO HIGH THAT IT FEELS
LIKE EXOME AND GENOME WILL COME
INTO PRACTICE.
AND CLEARLY ROOM FOR SYNERGIES
IN THE AREA OF PREGNANCY
MANAGEMENT IN THE PEOPLE WITH
AUTOIMMUNE CONDITIONS.
AGAIN, THANKS FOR YOUR
PRESENTATION.
>> PEDIATRIC PRESENTATIONS OF
DISEASE CAN BE SO INFORMATIVE
AND WE SEE IT IN PEDIATRIC FORMS
OF LUPUS WITH EARLY
PRESENTATIONS, SEVERE DISEASE
AND I AGREE THAT GENETIC
ANALYSIS IN THOSE POPULATIONS IS
LIKELY TO BE VERY INFORMATIVE.
COMPLETELY AGREE.
THANK YOU.
>> THAT'S MY FAVORITE PART OF
BEING PART OF COUNCIL IS
LEARNING ABOUT THE BREADTH AND
SCOPE ACROSS NIH AND THE
PARTNERSHIP.
AND THERE'S MANY TOUCH POINTS
BETWEEN I.C.s AND SHARED
EFFORTS, WHAT ARE YOUR THOUGHTS
IN HOW YOU ARE THINKING ABOUT
THE SHARING OR INTEGRATION OF
THE DATA ASSETS.
WE HEARD ABOUT AN EXAMPLE FROM
NICHD'S DATA WAREHOUSE, HOW ARE
YOU THINKING ABOUT
STRATEGICALLY?
>> THAT'S AN IMPORTANT TOPIC AND
ONE WE'VE BEEN TALKING ABOUT A
LOT AND HAS BEEN ON MY MIND
BEFORE I GOT TO THE NIH.
AN INSTITUTE HAS TWO COMPONENTS.
ONE THAT FOCUSES INTRAMURALLY TO
MAKE SURE OUR EFFORTS WITHIN THE
NIH ACROSS OUR I.C.s AND
CONSIDERING WHAT'S HAPPENING AT
THE OFFICE OF OUR DIRECTOR ARE
MAXIMALLY MUTUALLY INFORMATIVE
AND SET THEM UP FOR SUCCESS AND
COLLABORATION AND INTEGRATION.
THE STRUCTURES OF THE NIH CAN
PRESENT AN OBSTACLE TO
COORDINATION BUT THERE'S A LOT
OF INTEREST IN DOING EVERYTHING
WE CAN TO OVERCOME THAT.
WE HAVE A PILOT STUDY WE'VE
INITIATED IN OUR RFP TO UNDER
THE ISSUES.
WE LAUNCHED LAST WEEK A DATA
SCIENCE STRATEGY WORKING GROUP
OF OUR COUNCIL SO THEY CAN BE
FOCUSSING WITH THE PERSPECTIVE
ON THE EXTRAMURAL COMMUNITY,
WHAT THE EXTRAMURAL
INVESTIGATORS NEED, WHAT'S
HAPPENING AT THE NIH WE SHOULD
BE AWARE OF THAT CAN BE
LEVERAGED AND WHAT CAN WE AS
NIAMS, A RELATIVELY SMALL
INSTITUTE, WE'RE NOT A DATA
SCIENCE EXPERT BUT WHAT CAN WE
DO TO SUPPORT THE EXTRAMURAL
COMMUNITY AND IN THE CONTEXT OF
THE BROADER EFFORTS THE
ACCELERATING MEDICINES
PARTNERSHIP IS AN EXAMPLE IT'S
IMPORTANT WE THINK WITH THE
ISSUES AT THE OUTSET OF THE
STUDIES AS WE MOVE FORWARD WITH
EFFORTS IN AUTOIMMUNE DISEASE
POSITION OURSELVES TO SHARE DATA
ACROSS THESE BROAD CATEGORIES OF
CONDITIONS AND DISCOVER
RELATIONSHIPS THAT WE DIDN'T
THINK ABOUT AND WEREN'T ON OUR
RADAR SCREEN AND LIST OF
CANDIDATE SCREENS BUT CAN HELP
ACCELERATE PROGRESS IN THE
AREAS.
WE'RE ALREADY SEEING THROUGH THE
ACCELERATED MEDICINES
PARTNERSHIP MOLECULES POP UP IN
PA
PARKINSON'S AND THERE'S A GOAL
OF POSITIONING AND COORDINATING
EFFORTS SO THAT WE CAN LEARN AS
MUCH POSSIBLE ACROSS THE
DIFFERENT EFFORTS AS WE MOVE
FORWARD AND I HOPE WE CAN HAVE
THAT POINT OF VIEW MOVING
FORWARD AND HEAL AND COMMON FUND
PROJECTS TO MAXIMIZE OUR
INVESTMENT IN PROJECTS AND
ACCELERATE PROGRESS.
A BIG TOPIC.
>> IT IS.
THANK YOU FOR THE ANSWER.
>> BONNIE.
>> THANK YOU, DR. CRISWELL.
THAT WAS INFORMATIVE AND GREAT
TO HEAR A SHOUT OUT TO MY
COLLEAGUE, DR. SANDBORG HERE AT
STANFORD.
ONE QUESTION I HAVE FOR YOU IS
SPECIFICALLY AROUND THE ISSUE IS
IT'S IMPACTFUL TO PARTNER WITH
INTERNAL MEDICINE
RHEUMATOLOGISTS AND THE
DIFFICULTY POSTCOVID AND IN THE
LAST 10 YEARS IN RECRUITING
PEDIATRIC SCHOLARS INTO
SPECIALTIES FOR A LOT OF REASONS
BUT IN PARTICULAR THE ABILITY TO
GET TRACTION AND PARTNERING WITH
INTERNAL MEDICINE.
MAYBE I MISSED IT BUT ARE THERE
FOR EXAMPLE, TRAINING GRANTS AND
OTHER MECHANISMS IN PLACE WHERE
THEY CAN ENCOURAGE PEDIATRIC
RHEUMATOLOGISTS AND ENCOURAGE
SCHOLARSHIP IN THE PEDIATRIC
REALM.
>> UCSF IS FOR THE ENOUGH TO
HAVE A PEDIATRIC RHEUMATOLOGY
TRAINING PROGRAM AS WELL AS AN
ADULT RHEUMATOLOGY TRAINING
PROGRAM.
WE MADE EVERY EFFORT POSSIBLE TO
COLLABORATE.
IT WAS A WIN-WIN RELATIONSHIP.
SURE, THE ADULT RHEUMATOLOGY
PROGRAM AND THERE'S MORE
PATIENTS, THERE'S MORE SOLID
BASE OF FUNDING, MORE
INVESTIGATORS IN THIS AREA.
WE HAVE A LOT TO SHARE IN TERMS
OF INFRASTRUCTURE, TRAINING
OPPORTUNITIES TO BE
DERMATOLOGISTS BUT WE HAVE A LOT
TO LEARN AND PEDIATRIC
PRESENTATIONS OF DISEASE AND
THINKING STRATEGICALLY IN TERMS
OF FUND RAISING EVEN.
A PARTNERSHIP BETWEEN PEDIATRIC
AND ADULT SPECIALISTS IS A
WIN-WIN PROPOSITION.
UNFORTUNATELY, NOT ALL
INSTITUTIONS FROM THE
INSTITUTIONAL STANDPOINT ARE IN
A POSITION TO BE ABLE TO DO THAT
BUT ONE OF THE REASONS I'M
INTERESTED IN SUPPORTING
PEDIATRIC RESEARCH AND
PARTNERING WITH THESE
ORGANIZATIONS IS SO WE CAN HELP
THEM.
THE QUESTION I BRING IS HOW CAN
WE HELP YOU MAKE MORE
SUCCESSFUL?
AND WHAT CAN WE PROVIDE TO HELP
YOU BE MORE SUCCESSFUL.
THOSE CONVERSATIONS IN THE
CURRENT ROLE ARE AT THE EARLY
STAGES BUT I THINK IT WILL BE
MUTUALLY BENEFICIAL TO PROGRESS
IN THESE DISEASES.
I LOOK FORWARD AND OTHER IDEAS
YOU ALL HAVE ABOUT WHAT WE AS AN
INSTITUTE CAN BE DOING TO
SUPPORT TRAINING IN THE
PEDIATRIC POSITIONS.
WE WELCOME YOUR SUGGESTIONS.
>> THANK YOU, BONNIE AND
LINDSEY.
I THINK THERE'S AN AREA YOU MAY
NOT BE AWARE OF WHERE WE HAVE AN
INTEREST IN OUR STRATEGIC PLAN
IN THE TRANSITION OF CHILDREN
WITH CHRONIC DISEASES.
CHILDREN WITH RHEUMATOID
ARTHRITIS AS THEY TRANSITION TO
ADULT CARE AND DIFFICULTIES AND
CHALLENGES THEY FACE WHEN
THEY'VE BEEN IN A VERY
SUPPORTIVE ENVIRONMENT AND NOW
THEY HAVE TO GET A WHOLE NEW SET
OF PROVIDERS.
WE RECENTLY HAD A WORKSHOP
ADDRESSING THAT.
WE ALSO HAVE IN OUR STRATEGIC
PLAN PLACED A FOCUS ON
ADOLESCENTS BECAUSE THAT'S AN
AREA WHERE ACROSS NIH THERE
HADN'T BEEN A LOT OF ATTENTION.
THOSE MAY BE FUTURE AREAS WHERE
WE CAN WORK TOGETHER.
>> I'M HAPPY TO HEAR THAT.
REFERRING BACK TO UCSF WE HAD A
CURRENT AND TRANSITIONAL
CLINICAL WE THOUGHT WERE
IMPORTANT SO PATIENTS DON'T FALL
OFF THE RADAR SCREEN AND LOSE
CARE, SPECIALTY CARE WHEN THEY
HAVE OTHER CHALLENGES.
I'D LOVE TO TALK MORE ABOUT THAT
AND HOW WE CAN LEARN FROM EACH
OTHER AND HOW TO DO THAT AND
SUPPORT THAT.
IT'S REALLY IMPORTANT.
>> THIS WAS A FANTASTIC TALK.
YOU REALLY PAID ATTENTION TO OUR
REQUEST.
MORE SO THAN WE EXPECTED.
AND IT WAS A PLEASURE TO HEAR
FROM YOU.
I'LL TURN THE VIRTUAL MICROPHONE
TO DR. RASOOLY FOR OUR NEXT SET
OF INSTRUCTIONS.
>> THANK YOU.
I'M LOOKING AT MY WATCH AND
WE'RE ACTUALLY RIGHT ON TIME.
WE MAY BE A LITTLE BIT AHEAD OF
TIME.
LET'S TAKE A BREAK AND LET'S
REJOIN AT 2:35.
ABOUT 25 MINUTES.
2:35, ALL RIGHT?
THANK YOU SO MUCH.
>>WELCOME BACK.
I JUST WANTED TO WARN YOU THAT
WE HAVE A FAIRLY FULL AGENDA
THIS AFTERNOON SO THERE'S A
POSSIBILITY WE MAY END SLIGHTLY
AFTER FIVE THOUGH WE'RE FIVE
MINUTES AHEAD OF SCHEDULE RIGHT
NOW.
MAYBE AS THE NOT GOING TO BE THE
CASE.
TODAY IT'S MY GREAT PLEASURE TO
INTRODUCE DR. LE PICHON.
SHE'S AN INVESTIGATOR IN THE
DEVELOPMENT OF NEURODEGENERATION
AND HAS BEEN WITH US SINCE 2016.
HER LABORATORY EMPLOYEES MOUSE
GENETICS AND SCANNING OF THE
WHOLE BRAIN AND INVESTIGATING
THE EARLY EVENTS WITH THE ONSET
AND NEURODEGENERATION OF DISEASE
AND HER REPORT WILL BE FOLLOWED
BY OUR VOICE OF THE PARTICIPANTS
SESSION WHERE WE WILL MAKE THE
CONNECTION BETWEEN HER RESEARCH
AND A PERSON AFFECTED BY A
NEURODEGENERATIVE DISORDER
>> THANK YOU VERY MUCH.
I'M A TENURE TRACK INVESTIGATOR
AT NICHD AND I'VE BEEN HERE
SINCE 2016 TODAY I THOUGHT I'D
SHARE A SPECIFIC PROJECT NOT
REALLY A GENERAL OVERVIEW OF MY
PROGRAM BUT RATHER A PROJECT AS
THE SPECIFIC TO RELATED TO THE
VOICE OF THE PARTICIPANTS.
THIS HAS BEEN A PARTICULARLY
FRUITFUL AND INTERESTING
COLLABORATION WITH OTHER
INVESTIGATORS AT THE NIH AND AT
UNIFORM HEALTH SERVICES ACROSS
THE STREET.
THE TITLE OF MY TALK IS
PRECISION MOUSE MODELS OF
CHILDHOOD ALS CAUSED BY
EXCESSIVE SPHINGOLIPID
SYNTHESIS.
YOU'LL HEAR MORE ABOUT THE
PATIENT SIDE DIRECTLY AFTER.
WE STUDY NEURODEGENERATION
BROADLY.
WE'RE INTERESTED IN COMMON
MECHANISMS UNDERLYING
NEURODEGENERATION AND PATHWAYS
FOLLOWING NERVE INJURY AND
TRAUMATIC BRAIN AND NERVE
INJURIES.
WE HAVE MOUSE MUTATION AND STUDY
MOUSE INJURY STUDYING THE MOUSE
MODELS AND WE'RE INTERESTED IN
CELL SPECIFIC RESPONSES AND LOOK
AT CELL TECHNOLOGIES.
ANOTHER MODEL WE ARE ALSO USING
IN THE LAB NOW ARE HUMAN IPSC
DERIVED NEURONS TO STUDY
TRANSLATABILITY OF OUR FINDINGS
IN HUMAN CELLS AND HAVE A
TRACTABLE SYSTEM.
IN SIMPLE PERMS NEURO GENERATION
CAN BE OF HAPPEN DURING THE LIFE
SPAN AND WE STUDY
NEURODEGENERATION AND INTERESTED
HOW SPECIFIC MUTATIONS MAY LEAD
TO A DYSFUNCTION AND
DEGENERATION OF THE NERVOUS
SYSTEM.
WITH TODAY'S TECHNOLOGY MAKING
PRECISION MOUSE MODELS USING
CRISPR CAS 9 TECHNOLOGY IN MANY
CASES WHERE WE KNOW OF MUTATIONS
THAT CAUSE NEURODEGENERATION THE
GENE IS WELL CONSERVED IN THE
MOUSE.
WE CAN EDIT THE BASES THAT
CORRESPOND TO THE HUMAN MUTATION
AND STUDY AND CHARACTERIZE WHAT
PATHOLOGY DEVELOPS AND USE THE
WONDERFUL NEW TOOLS WE HAVE AT
OUR DISPOSAL IN NEUROSCIENCE
TODAY.
WHERE HE CAN LOOK AT THE
PATHWAYS THAT LEAD TO MUTATION
AND DISEASE AND IDENTIFY WHICH
CELL TYPES ARE INVOLVED AND
IDENTIFY POTENTIAL TARGETS.
IF WE COME UP WITH GOOD
PHENOTYPES IN THE MOUSE MODELS
AMENABLE TO THERAPIES THERE'S A
STEP TO GO ON TO TREAT HUMANS
BUT IT'S A GOOD FIRST STEP AND
IN MANY CASES WE DON'T HAVE
OTHER WAYS OF STUDIES WHAT WHAT
HAPPENS EARLIER IN THE DISEASE
PROCESS OR PRECEDING ONSET OF
SYMPTOMS.
I DO THINK THE MOUSE MODELS CAN
BE INCREDIBLY VALUABLE.
SO WHAT WE SET OUT TO DO AND
THIS WAS EARLY ON WHEN I JOINED
NICHD, I HEARD OF ONGOING WORK
ONE AT NINDS AND ONE AT NIA AND
ALL COLLABORATIVE EFFORTS BUT
THIS IS WHERE THE WORK OF
CLINICIAN COLLEAGUES OF MINE AND
SO AT NIMDS WE HAD DR. MOHASSEL
AND THE GROUP OF BRIAN TRAINOR
AT NIA AND THEY HAVE NOVEL
MUTATIONS IN THE GENE SPCTL1
THAT ARE ASSOCIATED WITH
CHILDHOOD ONSET ALS.
THESE ARE THE FIRST MONOGEN IT
METABOLOMIC MUTATIONS ASSOCIATED
WITH ALS.
A BRIEF DESCRIPTION OF WHAT IS
ALS, IT'S A FATAL
NEUROGENERATIVE DISEASE WHERE
THE PERSON'S BRAIN LOSES
CONNECTION WITH THE MUSCLES AND
LOSE THE ABILITY TO WALK, TALK,
EAT AND EVENTUALLY BREATHE.
THE ONSET CAN STRIKE ANY TIME.
WHAT'S PARTICULARLY INTERESTING
WHEN I LEARNED ABOUT THE CASES
IS THIS WAS REALLY A JUVENILE
ONSET KIND OF ALS WHICH WE THINK
MUST INVOLVE NEURODEVELOPMENTAL
COMPONENT IN ADDITION TO PERHAPS
THE LATER PROGRESSIVE
NEURODEGENERATION AND AS
MENTIONED BEFORE WHAT WAS
STRIKING ABOUT THESE CASES IS
THEY'RE THE FIRST DESCRIBED TO
METABOLOMIC MUTATIONS ASSOCIATED
WITH ALS.
FIRST I'LL GIVE BACKGROUND ON
THE BIO CHEMICAL PATHWAY AND
INTRODUCE THE MOLECULES INVOLVED
AND THEN I'M GOING TO SHOW YOU
SOME OF THE DATA FROM OUR MOUSE
MODELS.
THIS IS INITIATED BY THIS ENZYME
AND IT'S A SUBUNIT OF THIS
COMPLEX.
SPT CATALYZES THE CONDENSATION
OF SERINE AND HAS A LIPID FROM
WHICH ALL OTHERS ARISE.
AND SO THEIR LONG-CHAIN BASES
AND THESE ARE ESSENTIAL LIPIDS
OF MEMBRANES IN MAMMALIAN CELLS
AND HAVE A WIDE SPECTRUM OF
FUNCTION.
THEY'RE INVOLVED IN CELL GROWTH,
ADHESION, MIGRATION, CELL DEATH
AND YOU NAME IT.
SPT ACTIVITY REQUIRES CLOSE
HOMEOSTATIC REGULATION.
FIRST YOU NEED TO MAKE SURE
YOU'RE MAKING ADEQUATE
SPHINGOLIPID AND WANT TO AVOID
TOXICITY SO THE LEVELS HAVE TO
BE REGULATED.
THROUGH SENSING LEVELS OF DOWN
STREAM SPHINGOLIPIDS IN
PARTICULAR THE CERAMIDES AND
THIS COULD BE IMPORTANT TO WHAT
I'LL SHOW YOU LATER ON.
THIS IS A LITTLE BIT MORE IN
DETAIL FOR PATHWAY WITH THE
NAMES BUNT A FEW CHEMICAL
STRUCTURES TO SITUATE YOU.
CERAMIDES HAVE BEEN LINKED TO
SIGNALLING CELL DEATH.
WE HAVE IMPORTANT COMPONENTS OF
THE MYELIN SHEATH AND THE
CHEMICAL STRUCTURES THERE'S
VARIOUS MODIFICATIONS ON EVERY
RADICAL GROUP AND THERE COULD BE
DISTINCT BIOLOGICAL FUNCTIONS
WHICH OF YET TO BE UNCOVERED AND
BACK TO THE SPL GENE THE FIRST
STEP IN THE PATHWAY.
PREVIOUSLY NO MUTATIONS IN THIS
GENE ACTUALLY CAUSED HEREDITARY
AND SENSORY HSAN 1 AND THE
LOCATION OF OF THE MUTATIONS I'M
SHOWING IN THE DIAGRAM AND
PREVIOUS HAS WORK WHEN IT'S
MUTATED IN ANY OF THESE
LOCATIONS ASSOCIATED WITH THE
HSAN 1 SYNDROME, ONE OF THE MAIN
DRIVERS OF DISEASE IS THAT THE
MUTATION CAUSES SPT TO CHANGE
AFFINITY AND THIS CAUSES THE
PRODUCTION OF THE LIPIDS OPPOSED
TO THE DEOXYSPHINGOLIPIDS AND
IT'S BEEN CATEGORIZED IN THE
LAST FEW DECADES BUT THE
CHILDHOOD MUTATIONS THAT HAVE
BEEN LINKED TO SPLTC1 CLUSTER IN
THE DIFFERENT REGION OF THE
PROTEIN.
THIS WAS INTERESTING BECAUSE
THEY'RE ALL CLUSTERING IN THE
DOMAIN SHOWN IN RED AND HERE'S
AN EXAMPLE.
WORK FROM A GROUP SHOWED IN
PATIENTS WITH THESE MUTATIONS,
WE DON'T REALLY DETECT AN
ELEVATION IN THE
DEOXYSPHINGOLIPIDS AS WHAT IS
DETECTED IN THE HSAN PATIENTS
WHO HAVE THIS NEUROPATHY TYPE OF
PHENOTYPE.
INSTEAD, THERE WAS A DIFFERENT
HYPOTHESIS FOR WHAT THE
MUTATIONS ASSOCIATED WITH ALS
ARE DOING TO THE MOLECULE.
THIS IS GREATLY AIDED BY THE
RECENT CRYO EN STRUCTURE AND TWO
STUDIES PUBLISHED LAST YEAR
SHOWING THE EN STRUCK TEAR OF
SPTLC AND HIGHLIGHTS HOW THE ALS
VARIANTS AND SPTLC1 CLUSTER
HERE.
THE YELLOW IS SPTLC1 AND THE
ENZYME RESIDES AND YOU CAN SEE
SOME OTHER MUTATIONS HERE IN THE
LIGAND BINDING DOMAIN AND THE
ALS LINKED ONES ARE IN THE TRANS
MEMBRANE DOMAIN.
THIS IS EXACTLY WHERE THE
NEGATIVE REGULATOR GENE
INTERACTS.
IT'S THOUGHT THIS FACTOR
COMPETES WITH SUBSTRATE BINDING.
IF THESE RESIDUES ARE MUTATED IT
CAN REDUCE THE AFFINITY AND
REALLY ALTER THE NEGATIVE
REGULATION OF THE COMPLEX SO THE
COMPLEX AND THE ENZYME WOULD BE
OVERPRODU
OVERPRODUCING LIPIDS.
HERE'S THE ALTERNATIVE
HYPOTHESIS THAT THESE MUTATIONS
CAUSE AN INCREASE IN THE CAN
CANONICAL PRODUCTS OF THE
PATHWAY AND THE HYPOTHESIS WE
HAD GOING INTO THE MICE IS IT
THAT MICE ENGINEERED WITH THESE
ALS MUTATIONS SHOULD EXHIBIT
ELEVATED SPHINGOLIPID LEVELS AND
SIGNS OF NEURODEGENERATION.
WE MODELLED THAT AND I'LL
PRESENT UNPUBLISHED WORK FROM A
TERRIFIC GRADUATE STUDENT AND WE
WE HAVE DONE IS LOOK AT THE TWO
MUTATIONS THE A20S AND THE
DELETION OF THE AMINO ACIDS 40
AND 41.
A SIX-PAIR DELETION A
SINGLE-BASED MUTATION.
IF YOU LOOK IN THE THE ALIGNMENT
OF MAN AND MOUSE AND THIS
RESIDUE IS HIGHLY OBSERVED AS
ARE RESIDUES 40 AND 41.
AND SO WHAT WE DID WAS MADE AN
A20S MOUSE AND DELETION OF 40
AND 41.
SO JUST A KIND OF BRIEF OVERVIEW
BECAUSE THERE'S TOO MUCH DATA TO
SHARE IN THE SHORT TIME AND HAVE
A SUMMARY.
IN THE A20S LINE, WE FIND THAT
THE ANIMALS HAVE A NORMAL LIFE
SPAN AND THEY CAN BREED NORMALLY
AND BREED THEM TO HOMOZYGOTITY
AND WON'T HAVE A LOSS OF THE
MOTOR NEURON CELL BODIES.
THIS IS DIFFERENT THAN SOME
MOUSE MODELS OF ALS IN WHICH YOU
CAN REALLY OBSERVE A LOSS OF
MAYBE 50% OF THE MOTOR NEURONS
IN THE SPINAL CORD.
AND HOMEOZYGOTES HAVE THE MOST
AND SOME ANIMALS ARE VERY SMALL
WHEN BRED TO HOMO ZYGOCITY AND
THERE'S OBVIOUS MISDEVELOPMENT
OF THE NERVOUS SYSTEM AND
NEURODEGENERATION BUT THIS
CHARACTERIZATION IS ONGOING.
MOST OF WHAT I'LL SHOW IS DATA
THE LINE ON THE LEFT.
THIS IS JUST TO SHOW YOU THAT IN
BOTH FEMALE AND MALE MICE THE
HOMOZYGOUS AND HETEROZYGOUS
ANIMALS IN GREEN AND RED HAVE
SLIGHTLY LOWER BODY WEIGHT THAN
THEIR WILD TYPE COUNTERPARTS.
HERE ARE MEASUREMENTS AND WHAT
MY LAB DOES IS THEY'VE HARVESTED
DIFFERENT TISSUES FROM THE MOUSE
AND THEY CAN MEASURE THE
SPHINGOLIPIDS AND WE FOUND
THERE'S A DOSE-DEPENDENT
INCREASE IN VARIOUS
SPHINGOLIPIDS DOWN STREAM OF SPT
ACTIVITY THAT SHOWING IF MICE
CARRY ONE OR TWO COPIES OF THE
MUTATION IT'S IN LINE WITH WHAT
OTHER GROUPS HAVE FOUND IN THE
PATIENT COHORT.
INCREASE IN THE SPHINGOLIPIDS IN
THE CANONICAL PATHWAY.
AND WHAT ZOE HAS DONE IS
ANALYZED THE MICE AND WE CAN
FOLLOW THEM LONGITUDINALLY
OPPOSED TO WHERE WE HARVEST TISH
IT'S A TERM TERMINAL IF HE TURE
AND WHEN HERE I'M GOING TO SHOW
A MYELIN STAIN WHICH SHOWS THE
MYELIN ARE THE LIPID LAYERS THAT
ARE WRAPPING AROUND AXONS THAT
ALLOW FAST AXON CONDUCTION AND
AT 9 WEEKS OF AGE WE CAN TELL
THERE'S A SEVERE PHENOTYPE IN
THE HOMOZYGOUS ANIMALS AND
SOMETHING INTERMEDIATE IN THE
HOMO ZYGOTE.
THERE'S A LOSS OF INTENSITY OF
THE AXONS OVER ALL AND HERE YOU
SEE LIGHT GRAY AREAS AND WE SEE
OVER TIME THAT THE MYELIN LAYER
ITSELF SO TYPICALLY IN THESE
TISSUES FOR THOSE WHO AREN'T
USED TO SEEING THIS YOU SEE A
CROSS-SECTION THROUGH THE NERVE
AND THE SMALLER SAMPLES ARE
SMALLER DIAMETER AXONS AND THE
LARGER ONES THAT APPEAR WHITE
HAVE AYER RING OF MYELIN AROUND
IT AND THIS IS THE NORMAL
SITUATION AND ALLOWS LARGE
CALIBER AXONS.
THAT SEEMS TO BE PROGRESSION IN
THE OLDER ANIMALS AND THE LARGER
AXONS AND COMPARATIVELY THINNER
MYELIN AND A SIMILAR CALIBER
AXON.
ZOE WITH ANOTHER STUDENT IN THE
LAB MEASURED THE G RATIO
ESSENTIALLY A REFLECTION OF HOW
WELL MYELINATED THE AXONS ARE
AND THEY SHOW MORE POORLY
MILENATE -- MYELINATED THAN THE
SHOULD BE.
THIS IS A SUPER CLOSE UP OF SOME
OF THE AXONS SURROUNDED BY THEIR
MYELIN SHEATH.
IT'S PRODUCED BY A TYPE OF CELL
CALLED THE SCHWANN CELL AND HERE
YOU CAN SEE THESE LOOK FONORMAL
AND THE SHEATH AND THE
ASSOCIATED AXON.
HERE IN THE MUTANT YOU CAN SEE
HERE'S AN EXAMPLE OF PRETTY
THICK MYELIN SHEATH WITH ALMOST
NO MYELIN IN IT AND LOTS OF
ACCUMULATIONS IN THE SCHWANN
CELL ITSELF AND GAPS AND
EXCESSIVE WHAT LOOKS LIKE MYELIN
AND WE NEVER SEE THESE KINDS OF
IMAGES IN A WILD TYPE.
RARELY DO WE SEE IT IN THE
HETEROZYGOUS ANIMALS BUT WE
KNOWS THERE'S SOME OCCURRENCES.
SO IT PRODUCES PATHOLOGY IN THE
SCHWANN CELL AND HERE'S NEITHER
EXAMPLE OF THE GAPS THAT WE SEE
IN THE SCHWANN CELL AND
TYPICALLY WE ONLY HAVE ONE
MYELIN SHEATH AND ONE AXON
INSIDE THE SCHWANN CELL BUT HERE
YOU CAN SEE MULTIPLE SHEATHS AND
IT LOOKS LIKE A SIGN OF NERVES
TRYING TO REGENERATE.
YOU CAN SEE SOME SPROUTING HERE.
ALWAYS ON THE LEFT THE WILD TYPE
HEALTHY CONTROL AND ON THE RIGHT
THE HOMEOZYGOUS MUTANT AND THIS
IS MORE APPROPRIATELY MYELI
MYELINATED AND MATERIALS INSIDE
THE AXON AND THIS IS SHOWING IN
THE AXONS AND NEURONS THERE'S
DEFICITS NORMALLY YOU DON'T HAVE
A TRAFFIC JAM SUCH AS THESE
CELLS TRAPPED IN ONE PLACE.
SO ONE THING ZOE HAS ATTEMPTED
TO DO NOW AND THAT'S A SNAPSHOT
AND WE ATTEMPTED TO FOLLOW THE
ANIMALS OVER TIME USING AN
ELEC
ELECTROPHYSIOLOGICAL READ OUT
AND THE ANIMALS ARE ANESTHETIZED
AND ELECTRODES ARE PLACED ON THE
ANIMAL TO RECORD ACTIVITY THAT'S
ELICITED BY ELECTRODES THAT
STIMULATE THE NERVE AND ACTIVITY
IS RECORDED IN THE MUSCLE
RECEIVING INPUT FROM THE NERVE.
WHAT YOU MIGHT SEE IN A HEALTHY
ANIMAL IS A COMPOUND POTENTIAL
THAT LOOKS LIKE THIS AND I'M
GOING TO SHOW YOU IN THE NEXT
GRAPH THE BLUE MEASUREMENT AND
THE TOTAL AMPLITUDE.
THERE'S AN ARTIFACT STIMULUS AND
RESPONSE FROM THE MUSCLE.
LIKE A RESPONSE FROM MANY AXONS
FEEDING INTO THE MUSCLE AND WHAT
YOU MIGHT SEE IN A MOUSE MODEL
OF ALS IS A REDUCTION IN THE
AMPLITUDE.
AND IT CAN REFLECT MANY
DIFFERENT THINGS THE READOUT
TELLS YOU FOR EXAMPLE IT'S
REDUCED BUT DOESN'T TELL YOU
IT'S BECAUSE THERE'S LESS
NEURONS JUST SURVIVING OR THERE
ARE SAME NUMBER OF NEURONS BUT
MAYBE NOT AS WELL CONNECTED OR
THEY'RE NOT ABLE TO SIGNAL
PROPERLY.
THIS IS THE RESULT.
ZOE DID THE STUDY BLINDED AND
HAD WILD TYPE AND HETEROZYGOUS
ANIMALS AND SOME WERE STABLE AND
THIS IS RECORDED FROM A MUSCLE
CALLED THE TIBIALIS ANTERIOR AND
YOU CAN SEE IN GREEN THE
HOMOZYGOUS ANIMALS WHERE THEY
START OFF LOOKING RELATIVELY
NORMAL, THEY DROP OFF TO ABOUT
BETWEEN 30% AND 50% OF THE CMAP
AMPLITUDE YOU CAN CONTROL AND
THIS SEEMS TO PLATEAU WHERE YOU
GET CELL DEATH AND THIS WOULD
PLUMMET EVEN FURTHER AND WOULD
KIND OF BOTTOM OUT BETWEEN 10
AND 20 MINUTE VOLTS.
THIS IS SOMETHING INTERMEDIATE
AND THE TYPES OF MODELS WE HAVE
LOOKED AT IN THE LAB BUT
NONETHELESS A SIGNIFICANT AFFECT
AND CORRELATES WITH THE
HISTOPATHOLOGY WE'VE BEEN SEEING
IN THE NERVE.
I WANT TO SHOW YOU ONE PIECE OF
DATA WE'RE QUITE EXCITED ABOUT.
WE'VE BEEN ABLE TO SERVE FROM
THE SERUM OF THESE ANIMALS, WE
CAN COLLECT BLOOD FROM THE
ANIMALS AND SHOWED YOU FROM THE
BLOOD WE CAN DETECT THE LIPIDS
AND THE SPHINGOLIPIDS AND HERE
WE CAN DETECT NEUROFILAMENT
LIGHT AND IT'S A BIOMARKER OF
NEURODEGENERATION AND A
CYTOEXCY
CYTOSKELETAL PROTEIN EXPRESSED
IN NEURONS WITH LONG AXONS SUCH
AS MOTOR AND SENSORY NEURONS AND
DETECT HIGHER THAN NORMAL LEVELS
AND THERE'S A MARKER OF
NEURODEGENERATION.
THIS IS A DIFFERENT AGE OF THE
ANIMAL OVER TIME.
YOU CAN SEE ALREADY AT A YOUNG
AGE WE'RE DETECTING HIGHER THAN
NORMAL LEVELS OF NEUROFILAMENT
LIGHT.
HERE YOU'RE SEEING THE LEVELS OF
NEUROFILAMENT LIGHT AND THE HOMO
ZYGOTES IS HIGHER AND INCREASES
WITH AGE.
AND IN A PREVENTION TREATMENT
STUDY YOU COULD MEASURE LEVELS
BEFORE AND AFTER PERIOD OF SOME
KIND OF TREATMENT WE MIGHT WANT
TO TEST IN THE ANIMALS TO REDUCE
THE LEVELS OF SPHINGOLIPIDS.
I JUST WANT TO SHARE TWO SLIDES
ON THE SECOND MOUSE MODEL WE'VE
BEEN DEVELOPING BECAUSE IT'S
ALWAYS USEFUL TO IF YOU'RE
MODELLING A DISEASE IN MOUSE AND
YOU SEE SIMILAR THINGS WITH A
SERIES CAN INCREASE THE POWER
FOR YOUR ANALYSIS AND
OBSERVATION AND UNDERSTANDING
FOR WHAT'S HAPPENING IN THE
SYNDROME.
WITH THIS DELETION OF THESE
AMINO ACIDS 40, 41 ARE SMALLER
AND A TYPICAL ANIMAL MOUSE AT 4
TO 5 WEEKS ARE ABOUT 15 GRAMS AN
THESE ARE CLOSER TO 5.
THEY'RE TEENY.
IN FACT THEY ONLY SURVIVE TO
ABOUT 5 WEEKS OLD.
IT'S BEEN TRICKIER TO STUDY
BECAUSE WE HAVE THIS RELATIVELY
SHORT WINDOW IN WHICH TO DETECT
AND COLLECT THESE ANIMALS.
WE'RE CURRENTLY INVESTIGATING
THE PATHOLOGY AND STILL
INVESTIGATING WITH PERHAPS THE
HETEROZYGOTE AND YOU SEE IN THE
HOMO ZYGOTE IT LOOKS QUITE
DIFFERENT FROM THE WILD TYPE AND
THE HETEROZYGOUS IS IN BETWEEN.
THIS IS 4 WEEKS OLD AND DURING
THE PERIOD STILL POST-NATALLY OR
LATER DEVELOPMENT YOU CAN SEE
THE MYELIN HAS NOT FULLY
DEVELOPED HERE OR NOT PROPERLY
DEVELOPED IN THE HETERODIE --
HETERODIE GOUS ANIMALS AND YOU
CAN SEE THIS IS ONLY PARTIAL AND
YOU CAN SEE YOU MAY BE ABLE TO
RECOGNIZE THE PATHOLOGIES I
SHOWED IN THE SCHWANN CELLS IN
THE LIGHT GRAY.
WE'RE SEEING A
NEURODEVELOPMENTAL ISSUE
ASSOCIATED WITH THIS MUTATION AS
WELL PROGRESSIVE DEGENERATION
AND HAVE YET TO UNPACK WHY
THEY'RE SO DIFFERENT BETWEEN THE
TWO ANIMAL BUT DO SEE ELEVATED
SPHINGOLIPIDS IN THE OTHERS AND
WE HAVE A MOUSE FOR COMPARISON
AT THE YOUNG AGE OF 4 TO 5 WEEKS
OLD WHERE WE HAVE A SMALL
ELEVATION IN THE LINE BUT IT'S
HIGH IN THE DEL 40-41 HOMO
ZYGOTES AT THIS TIME AND THE
REASON WE WERE INTERESTED IN
THIS MUTATION IS IT'S A SIX-PAIR
DELETION.
THAT'S SOMETHING ONE CAN WORK
WITH TO TRY TO SILENCE A MUTATED
ALLELE.
I'VE BEEN SHOWING ALL THE DATA
SHOWING THE HOMOZYGOUS MICE HAVE
A SIGNIFICANT PHENOTYPE BUT
PATIENTS ARE CARRYING JUST ONE
OF THESE ALLELES AND THIS IS
TYPICAL IN THE WORLD OF MOUSE
MODELLING.
SOMETIMES YOU NEED MORE COPIES
OF THE MUTATED ALLELE TO SEE A
PHENOTYPE.
IN PEOPLE THEY CARRY ONE AND
THAT WOULD BE SUFFICIENT TO
CAUSE THAT SILENCE IT COULD
CAUSE THE ALLELE.
MUTATIONS ASSOCIATED WITH
JUVENILE ALS PRODUCE
NEURODEGENERATION PHENOTYPES IN
GENE-EDITED MICE.
WE HAVE ONGOING CHARACTERIZATION
GOING ON AND WE'RE PLANNING A
PREVENTION STUDY USING
INHIBITERS OF SPTLC1 IN THE
MOUSE MODEL I'VE DESCRIBED
TODAY.
WE HOPE TO DISCOVER A PHENOTYPE
IN THE MICE FOR FUTURE
TRANSLATIONAL STUDIES AND I WANT
TO HIGHLIGHT THIS RESEARCH CAN
HAVE RELEVANCE FOR POTENTIALLY
ALS PATIENTS AT LARGE OR IN THE
SPORADIC POPULATION FOR ALS
BECAUSE MUTATIONS HAVE BEEN
REPORTED IN SPORADIC ALS
PATIENTS THAT WOULD HAVE A MORE
ALS PROFILE.
THERE'S A POSSIBILITY OF
SPORADIC ALS AS WELL AS
POTENTIALLY USING DISEASE
SPHINGOLIPIDS AND I WANT TO
THANK PEOPLE AND THE GRADUATE
STUDENT IN MY LAB WHO
SPEARHEADED THIS WORK AND IT'S
BEEN A FANTASTIC COLLABORATION
WITH A LAB AT HSUHS AND THANK
YOU.
I'M HAPPY TO TAKE QUESTIONS.
WE'RE HOPING NOW FOR QUESTIONS
AND TURN OFF THE SLIDE TO SEE
EVERYONE.
>> THANK YOU FOR AN INTERESTING
PRESENTATION.
SINCE YOU FOCUSSED ON SCIENCE, I
HAVE SOME SCIENCE QUESTIONS FOR
YOU.
ARE YOU SUGGESTING THE SPCLTC1
GENE IS INTERRUPTING MYELIN
PRODUCTION.
IS THIS A FORM THAT MAY START
START WITH THE GLIAL FORM AND
WITH THE PATHOLOGY SHOULD
PROGRESSIVELY, IT SEEMS MORE
LIKELY IF YOU LOOK AT HIGH-LIMB
MOTOR NEURONS YOU WOULD START TO
SEE LOSSES.
HAVE YOU LOOKED AT THAT MOTOR
POPULATION AT LATE STAGES TO SEE
IF INDEED THEY DO DIE?
>> THESE ARE BOTH REALLY GREAT
QUESTIONS.
THANK YOU.
FOR THE FIRST QUESTION, WHETHER
WE THINK IT ALS CAN BE CAUSED BY
THE CELLS THERE'S EVIDENCE THAT
IT'S A LITTLE TRICKY.
I DON'T THINK WE CAN CONCLUDE
WITH THE ANIMAL MODEL WE MADE TO
REALLY SHOW IT WOULD BE
INTERESTING TO MAKE A
CONDITIONAL MODEL FOR EXAMPLE
JUST EXPRESS IT IN THE
MYELINATING CELLS BUT BASED ON
WHAT WE'VE SEEN, WE SEE EVIDENT
PATHOLOGY IN THE SCHWANN CELLS
AND I'D GUESS IT'S SIMILAR IN
THE CENTRAL NERVOUS SYSTEM BUT
WE ALSO SEE -- I FEEL LIKE THE
TYPES OF PATHOLOGY WE'RE SEEING
IN THE NEURONS WE ALSO SEE
SOMETHING THERE.
SPT PROBABLY HAS IMPORTANT ROLES
IN NEURONS AS WELL SO IT'S
DIFFICULT TO DISSECT THE TWO
EFFECTS BUT DEFINITELY THE MOST
STRIKING WHEN YOU JUST LOOK AT
THE IMAGES THE SCHWANN CELLS
LOOK SO INTRIGUINGLY DIFFERENT.
IT IS POSSIBLE MANY AXONAL
PATHOLOGIES CAN JUST BE A
CONSEQUENCE OF PROBLEMS WITH
MYELINATION FOR SURE SO I'M NOT
DISCOUNTING THE PATHOLOGY IN
NEURONS IS SECONDARY TO THE
MYELINATING CELL.
FOR THE SECOND QUESTION, HAVE WE
LOOKED IN HIGH LIMB MOTOR
NEURONS IN LATER STAGES?
I DIDN'T SHOW COUNTS TODAY
BECAUSE WE'RE STILL IN THE
PROCESS OF DOING THAT.
THERE'S NOT A HUGE OVERT LOSS
LIKE I MENTIONED.
IT'S NOT LIKE WE CAN LOOK AT ONE
IMAGE AND KNOW THE ANSWER.
WE HAVE TO DO QUITE DETAILED
QUANTIFICATION BUT WE DO SEE
LATER SIGNS OF MOTOR NEURON
DEGENERATION.
IT'S NOT MASSIVE.
I THINK I CAN SAY THAT WITH SOME
CONFIDENCE BECAUSE THE ANIMALS
LIVE NORMAL LIFE SPANS AND DON'T
DEVELOP SEVERE MOTOR SYMPTOMS
BUT I THINK THERE MIGHT BE SOME
MILD LOSS.
I THINK IN THE SECOND MODEL THE
MORE RAPID MODEL WE HAVEN'T
LOOKED AT THAT AT ALL AND IT
WILL BE INTERESTING TO DO SO.
>> IN THE INTEREST OF TIME I
THINK WE'RE GOING MOVE ON.
THANK YOU VERY MUCH, CLAIRE AND
THOSE FAMILIAR WITH OUR ADVISORY
COUNCIL MEETINGS YOU KNOW WE
REGULARLY HAVE A SEGMENT CALLED
VOICE OF THE PARTICIPANT AND
HEAR FROM THOSE WHO HAVE
PERSONAL EXPERIENCES FOR
RESEARCH THAT NICHD FUNDS AND WE
LOVE OUR MICE BUT WE WANT TO
HEAR HOW THE CONDITIONS AFFECT
HUMANS AS WELL.
WE ARE VERY FORTUNATE TO HAVE
MS. RIVKA HERZFELD JOIN AND I
SEE SHE'S JOINED ON AND WILL BE
JOINED BY DR. PAYAM MOHASSEL WHO
HAS NOW MOVED TO THE JOHNS
HOPKINS UNIVERSITY SCHOOL OF
MEDICINE.
DR. MOHASSEL IS A PHYSICIAN
SCIENTIST WITH A RESEARCH FOCUS
ON TRANSLATIONAL STUDIES IN
GENETIC NEUROMUSCULAR DISORDERS
AND HE WAS RECENTLY RECRUITED TO
JOHNS HOPKINS UNIVERSITY SCHOOL
OF MEDICINE AS AN ASSOCIATE
PROFESSOR OF NEUROLOGY AND LOOKS
AT RARE MUSCULODISEASES AND
PROOF OF CONCEPT STUDIES AND
EARLY PHASE CLINICAL TRIALS.
WELCOME, DR. MOHASSEL AND MS.
HEARST -- HERZFELD.
>> THANK YOU FOR THE OPPORTUNITY
TO TALK IN THE SPECIAL SESSION,
VOICE OF THE PARTICIPANT.
I THINK HOPEFULLY RIVKA WILL BE
ABLE TO JOIN AND A LOT OF THE
SCIENCE AND BACKGROUND I'M NOT
GOING SPEAK MUCH ABOUT THAT PART
AND START BY INTRODUCING RIVKA.
SO IT'S MY PLEASURE TO INTRODUCE
RIVKA.
SO YOU ALL KNOW A LITTLE BIT
ABOUT HER, SHE HAS A MASTER'S
DEGREE IN BIBLE STUDIES AND A
TECHNICAL WRITER BACK IN NEW
JERSEY AND SHE'S BEEN WORKING
HERE ABOUT THREE YEARS AND
BEFORE THAT WAS A TEACHER.
SHE IS VERY CURIOUS AND VERY
MOTIVATED TO UNDERSTAND WHAT
CAUSES WEAKNESS IN HER AND HER
FAMILY WHO CARRY THE SAME
MUTATION IN THE GENE, THIS PTLC1
AND I CAN SPEAK FROM PERSONAL
EXPERIENCE SHE'S VERY
PERSISTENT.
WE HAD KNOWN ABOUT RIVKA AND HER
FAMILY BUT DUE TO THE PANDEMIC
THEY DIDN'T HAVE THE OPPORTUNITY
TO COME TO THE NIH UNTIL LAST
YEAR AND HER AND HER SIBLINGS
HAVE THIS MUTATION AND AS SHE'LL
EXPLAIN THE DISEASE AFFECTS HER
AND HER FAMILY MEMBERS IN
DIFFERENT WAYS BUT I HOPE THAT
YOU WILL LEARN MORE ABOUT HER
EXPERIENCE AND HER STORY AND
HOPES FOR THE FUTURE AS SHE
TALKS TO US.
HI, RIVKA.
>> HI, EVERYONE.
>> CAN YOU TELL US A LITTLE BIT
ABOUT YOURSELF, YOUR FAMILY AND
HOW THIS DISEASE HAS AFFECTED
YOU AS YOU'VE GONE THROUGH
CHILDHOOD AND SO ON.
>> SURELY.
I REALIZED THERE WAS SOMETHING
WRONG WHEN I SAW MY SISTER AND I
WERE PLAYING IN THE BACKYARD AND
I NOTICED SHE WAS DOING WHAT TO
ME LOOKS LIKE A MOON WALK A LA
MICHAEL JACKSON WALKING ON HER
TOES AND I MENTIONED IT TO HER
AND SAID WHY ARE YOU WALKING
THAT WAY AND SHE SAID THAT'S HOW
I WALK.
WE WERE REALLY ACTIVE KIDS AS
YOU CAN SEE IN THE PICTURES, I
LIKED HORSEBACK RIDING AND WAS
ALWAYS ON THE PLAYGROUND AND I
TOOK TAEKWONDO FOR 10 YEARS AND
PLAYED SOCCER AND THINGS STARTED
TO GET MORE DIFFICULT FOR ME AND
I WASN'T GOING AS FAST AS I
WANTED TO GO AND I REMEMBER
THINKING DURING A RELAY RACE
THAT I WANTED MY BODY TO GO
FASTER AND IT WASN'T AND I
ALREADY UNDERSTAND A LITTLE BIT
FROM PBS PROGRAMMING THAT THE
BRAIN IS TALKING TO YOUR MUSCLES
AND YOUR MUSCLES PICK UP THAT
SIGNAL AND DO WHAT THEY'RE
SUPPOSED TO AND CONFUSED WHY
THAT HASN'T HAPPENING WITH ME.
I GOT MY SECOND-DEGREE BLACK
BELT AND USED TO PLAY THE DRUMS
AND THOSE ARE VERY ACTIVE THINGS
FOR YOUR MUSCLE GROUPS AND IT
BECAME MORE DIFFICULT FOR ME
OVER TIME.
IT WAS DEMORALIZING AND
FRUSTRATING SO I WANTED TO
FIGURE IT OUT.
>> THANK YOU FOR SHARING ALL
THAT.
CAN YOU ALSO TELL US A LITTLE
BIT ABOUT AS YOU NOTICE THE
WEAKNESS WAS PROGRESSING HOW
THAT MIGHT HAVE AFFECTED YOUR
RELATIONSHIP WITH OTHERS OR
OTHER FUNCTIONS THAT YOU
PARTICIPATED IN THROUGHOUT LIFE
THAT ARE IMPORTANT TO YOU?
>> SURE.
SO YOU HAD MENTIONED I'M A VERY
ACTIVE PERSON.
I'M ACTIVE IN MY SYNAGOGUE, I'M
AN ACTIVE VOLUNTEER IN MY
COMMUNITY AND COULDN'T DO THOSE
THINGS SO SEPTEMBER 2009 TO JUNE
2010 I WAS IN ISRAEL FOR A YEAR
OF STUDY ABROAD AND IN ISRAEL
THERE'S LOTS OF MOUNTAINS TO
HIKE.
IT WAS REALLY DIFFICULT FOR KNOW
HIKE UP ONE CERTAIN MOUNTAIN.
I REMEMBER CRYING ON THE BUS
BACK TO JERUSALEM SAYING TO
MYSELF, OH, NO, WHAT IF I HAVE
WHAT MY SIBLINGS HAVE SO CLEARLY
HAVE AS A MUSCLE PROBLEM.
AND WHEN I GOT BACK TO THE
UNITED STATES I DECIDED TO DIG
DEEPLY INTO FIGURING OUT HOW I
COULD AMELIORATE THAT PROBLEM.
I WANT TO SWITCH GEARS A LITTLE
BIT AND HEAR AS YOU WENT ON THIS
QUEST TO FIGURE OUT WHAT'S GOING
ON WITH YOU AND YOUR FAMILY, HOW
YOUR INTERACTIONS WITH MEDICAL
PROFESSIONALS, HOW LONG DID IT
TAKE, WHAT TYPE OF TESTING DID
YOU GO THROUGH OVER THE YEARS
UNTIL THE DIAGNOSIS WAS
EVENTUALLY IDENTIFIED.
>> IT'S BEEN A COUPLE YEARS.
I MEAN ABOUT 10.
ABOUT A DECADE.
MY SIB LINGS BECAUSE WE WERE THE
FIRST TO PRESENT WITH THIS
JUVENILE ALS THEY WERE SEEN BY
NEUROLOGISTS FIRST AND THEY WERE
TOLD THEY HAD A DIFFERENT TYPE
OF MUSCLE DISEASE THEN THINGS
WEREN'T MAKING SENSE IN TERMS OF
THE FACT THAT WE DON'T
EXPERIENCE PAIN AND WHEN I SAY
WE DON'T EXPERIENCE PAIN I MEAN
MY SIBLINGS ARE NOT IN CONSTANT
PAIN.
WE FEEL PAIN IF WE TOUCH
SOMETHING HOT OR PRICK OUR
FINGERS.
WE'RE NOT NEUROPATHIC BUT WE
DON'T FEEL A CONSISTENT PAIN.
SO THAT DIAGNOSIS DIDN'T MAKE
SENSE.
I STARTED TO REACH OUT TO
MEDICAL PROFESSIONALS.
I REACHED OUT TO A DOCTORATE
HACKENSACK MEDICAL CENTER AND
DID SEQUENCING WHICH TOOK ABOUT
A YEAR TO SEQUENCE MY WHOLE
GENOME AND THEY HAD INITIALLY
TOLD ME THAT THERE WAS A PROBLEM
ON MY A63 GENE AND A SAID
PERFECT, WE HAVE AN ANSWER.
AND IT MADE SENSE TO ME BECAUSE
IT'S A COLLAGEN GENE AND THAT
HELPS MAKE MUSCLES AND MUSCLE
TONE BUT THEN THE DOCTORS DID
MORE RESEARCH AND DECIDED IT WAS
A VARIATION ON THE SPCTL1 GENE
AND THAT'S WHEN DR. MOHASSEL
GOLD INVOLVED.
>> THAT'S RIGHT.
BY THE TIME WE WERE ABLE TO GET
IN TOUCH OR YOUR LOCAL DOCTORS
WERE ALREADY ON THE RIGHT TRACK
THROUGH A COLLABORATION WE
BECAME AWARE OF THAT MUTATION IN
YOUR FAMILY THROUGH THE
HACKENSACK GROUP.
AND THEY GOT YOU ALL DOWN HERE
AT THE NIH.
THE LAST OPEN QUESTION I WANTED
TO ASK YOU TO TELL US A LITTLE
BIT ABOUT HOW THINGS ARE GOING
FOR YOU RIGHT NOW.
WHAT ARE THE THINGS YOU'RE
INVOLVED IN AND HOPES FOR THE
FUTURE AND WHAT THE MEDICAL
RESEARCH CAN POTENTIALLY DELIVER
FOR YOU AND YOUR FAMILY.
>> YEAH, SURE.
SO RIGHT NOW MY HOPES FOR THE
FUTURE ARE PRETTY HIGH.
MAYBE NAIVE OF ME BUT I'M VERY
HOPEFUL BECAUSE EVEN 10 OR 20
YEARS AGO THE WHOLE GENOME
HADN'T BEEN SEQUENCED AND THEY
WOULDN'T HAVE BEEN ABLE TO PIN
POINT THE PROBLEM ON WHAT TYPE
OF GENE AND I GOT DETAILED
FASCINATING RESEARCH AND SO I
HAVE HOPES THE MEDICAL COMMUNITY
HAS AN INTEREST IN THIS BECAUSE
EVEN THOUGH THIS IS NOT A
TYPICAL TYPE OF ALS, IT IMPACTS
MY LIFE IN SUCH A GREAT WAY.
I CAN'T EXPERIENCE NATURE THE
WAY I'D WANT TO.
I CAN'T WORK OUT AND PLAY SPORTS
THE WAY I'D WANT TO AND I AM WHY
IT PA-- IT IMPACTS MY JEWISH LI
AND I CAN'T PARTICIPATE IN
SURVEYS ASPECTS OF JUDAISM AND
EVEN PRAYER A LOT OF SITTING AND
STANDING WHEN THE ARC IS OPEN
AND I UNDERSTAND BUT IT MAKES ME
FEEL UNCOMFORTABLE AND I TRY TO
TRY MY BEST AT EVERYTHING THAT I
DO AND ANYTHING I CAN DO TO HELP
THE MEDICAL COMMUNITY TO FIGURE
THIS OUT BECAUSE IF THERE IS A
SOLUTION, I WOULD LOVE TO HELP
BE PAERLT OF THAT.
-- AN APART OF THAT.
>> THANK YOU FOR ALL THAT.
AT THIS POINT WE CAN PAUSE ON
THIS.
DO YOU WANT TO SHARE ANYTHING
ELSE WITH EVERYONE?
>> I JUST WANT TO MAKE SURE I
DON'T PASS WHATEVER THIS ALS
GENOME TYPE IS.
I DON'T WANT TO PASS THAT DOWN
TO CHILDREN.
I KNOW THAT IS SLIGHTLY
COMPLICATED BECAUSE I WOULD NEED
TO MAKE AN EMBRYO AND IT WOULD
NEED TO BE TAKEN OUT AND I KNOW
THAT IS SOMETHING THAT IS SORT
OF COMPLICATED AT THIS POINT
BECAUSE EMBRYOS ARE SMALL AND
GENES ARE EVEN SMALLER IN THOSE
EMBRYOS.
I JUST HOPE I CAN GET THE LIFE
THAT I HAD BACK IN SOME
CAPACITY.
I'M NOT ASKING FOR IT ALL.
I DON'T KNOW -- EVEN IF THERE
WERE A DRUG OR A TREATMENT OR A
THERAPY OF SOME SORT, I KNOW
THAT I WOULDN'T BE ABLE TO GO
BACK TO TAEKWONDO AND HOPE TO
GET BACK TO AN ACTIVE LIFESTYLE
AND I KNOW I MAINTAIN AN ACTIVE
LIFESTYLE DESPITE MY PHYSICAL
CHALLENGES.
THAT'S MY HOPE.
>> THANK YOU VERY MUCH.
>> THANK YOU.
>> I THINK WE KAN OPEN THIS UP.
RIVKA, I'M DR. BIANCHI THE
DIRECTOR OF THE NICHD.
I WANT TO THANK YOU FOR BEING SO
OPEN AND HONEST.
I HAD A COUPLE QUESTIONS FOR
YOU.
I AM A REPRODUCTIVE GENETICISTS
IN YOUR CLINIC TRAINING AND I
SAW YOUR FATHER WAS ON AN
ASSISTED MOBILITY DEVICE.
IS HE ALSO AFFECTED?
>> INTERESTINGLY, MY FATHER IS
NOT IMPACTED BY THIS ATYPICAL
TYPE OF ALS.
MY DAD HAS AN IDIOPATHIC NEU
NEUROON -- NEUROPATHY AND I
DON'T KNOW TO WHAT EXTENT HIS
CONDITION IMPACTS OURS BUT EVEN
IF HE HAS THE SAME TYPE OF
NEUROMUSCULAR POSITION WE HAVE,
IT DOESN'T PRESENT THE SAME WAY.
>> HAS EVERYONE IN THE FAMILY
UNDER GONE THE GENETIC TESTING
NOW THAT WE KNOW ABOUT THE
SPCTL1 MUTATION?
>> NOT EVERYONE HAS BECAUSE I
THINK IT IS PRESUMED THAT FOUR
CHILDREN HAVE THE SAME PARENTS
THAN IT IS MOST LIKELY THE SAME
MALFUNCTIONING GENE THAT IS
CAUSING THIS ATYPICAL ALS.
>> IF I MAY JUST ADD TO THAT A
LITTLE BIT.
RIVKA'S FAMILY IS A LITTLE BIT
COMPLICATED AND WE DON'T
UNDERSTAND EVERYTHING FULLY
GOING ON BECAUSE HER FATHER
CARRIES THE EXACT SAME MUTATION
AS ALL THE SIBLINGS BUT PRESENTS
A SLIGHTLY DIFFERENT PHENOTYPE
AND HE HAPPENS TO HAVE A
SLIGHTLY DIFFERENT METABOLIC
PROFILE IN THIS SPHINGOLIPID
OMICS AND DON'T UNDERSTAND WHAT
MAY BE AT PLAY HERE BUT THE
WHOLE GENOME HAS HAD WHOLE EXOME
AND GENOME SEQUENCING AND THE
FATHER HAS A SLIGHT PHENOTYPIC
VARIABILITY.
>> WHAT I'M GETTING AT BECAUSE
YOU MENTIONED CHILDREN, WHETHER
THIS IS AUTOSOMAL DOMINANT
VERSUS RECESSIVE AND I THINK
YOU'RE SAYING DOMINANT.
50% OF CHILDREN OF AFFECTED
PEOPLE WOULD BE EXPECTED TO HAVE
THIS VARIATION IN THE GOON AND
YOU MIGHT SAY HOW COME IT'S 4
OUT OF 4.
UNFORTUNATELY GENETICS HAS NO
MEMORY SO EACH CONCEPTION IS A
NEW EVENT.
I DID WANT TO CORRECT ONE THING
YOU SAID, YOU SEEM TO IMPLY THAT
IN AN EMBRYO WE WOULD TAKE OUT
THE MUTATED GENE AND SO THAT IS
NOT WHAT WE DO.
RIGHT NOW WHAT WE DO, IN VITRO
FERTILIZATION AND ONLY TRANSFER
EMBRYOS WHO DON'T HAVE THE
MUTATION AND IT'S POSSIBLE FOR
ANYONE IN THE FAMILY WHO WANTS
TO PURSUE HAVING CHILDREN TO
TRANSFER UNAFFECTED EMBRYOS IF
THAT'S THE DESIRE.
>> SORRY.
>> THAT'S CLINICALLY AVAILABLE
RIGHT NOW.
>> OKAY, GREAT.
>> OTHER QUESTIONS?
>> THANK YOU FOR SHARING YOUR
SITUATION.
I HAVE A QUESTION IN TERMS OF
TECHNOLOGIES YOU MAY BE AWARE
THAT TO HAVE REGAINED MOBILITY
FOR EXAMPLE OTHER TECHNOLOGIES,
I WONDER IF YOU'RE AWARE OF THE
TECHNOLOGIES OR IF THERE'S
BARRIERS.
>> I'M AWARE OF EXOSKELETON I
THINK WHAT IF MY ANKLE ROLLS OUT
WHILE I AM WALKING AND NOT ONLY
WOULD I FALL BECAUSE IT HAPPENS
TO BE MY ANKLE IS THE IS THE
CAUSE OF MOST OF MY FALLING.
MY ANKLE ROLLS OUT TOWARDS THE
OUTSIDE OF THE HEEL AND THEN I
FALL AND THERE'S NOTHING I CAN
DO TO STOP IT.
IF I WERE TO FALL IN AN EXO
SKELETON WOULD I GET MORE HURT
AND I DO USE A SMALL SCOOTER SO
IT HAS A JOY STICK.
IT HAS A SPEED DIAL.
IT'S NOTHING FANCY.
MY BROTHER'S IS VERY FANCY.
IT CAN RAISE ITSELF AND LOWER
ITSELF, IT CAN LEAN BACK.
I DON'T HAVE ANYTHING LIKE THAT
AND THAT'S FINE.
I WAS ALSO TOLD BECAUSE THERE'S
SUCH GREAT MUSCLE WEAKNESS IN MY
LEGS AND THIGHS SPECIFIC, WHAT
IF MY THIGHS JUST GAVE OUT AFTER
BEING SO TIRED FROM WALKING AND
THEN WHAT GOOD WOULD IT DO?
>> HAVE YOU CONSULTED WITH A
SPECIALIST?
>> I HAVE NOT.
>> IT MAY NOT BE A SOLUTION BUT
IT WOULD BE GOOD TO LEARN MORE
ABOUT THE TECHNOLOGY AT LEAST TO
MITIGATE SOME OF THE FEARS YOU
HAVE.
>> THANK YOU.
>> RIVKA, YOU MAY OR MAY NOT
KNOW THE NIH HAS THE CENTER FOR
NATIONAL REHABILITATIVE RESEARCH
AND EXPERTS IN REHABILITATIVE
MEDICINE.
WE'RE HAPPY TO CONNECT YOU AS A
BENEFIT OF JOINING US.
>> I'M A REPRODUCTIVE E
ENDOCRINOLOGIST AND SOMETIMES
THERE'S A LOT YOU CAN LEARN TO
HELP EMPOWER YOU AND IF YOU'RE
IN NEW YORK I KNOW LOTS OF
REPRODUCTIVE ENDOCRINOLOGISTS IN
NEW YORK.
IF WE CAN HELP YOU MAKE YOUR
LIFE BETTER I DON'T KNOW ANYONE
WOULDN'T WANT TO DO SO AND IF
THERE'S A WAY TO SHARE
INFORMATION, PLEASE LET US KNOW.
>> THANK YOU.
WE'LL FOLLOW-UP ON SOME OF THESE
RESOURCES.
WE'LL TELL YOU WHERE THEY ARE
AND IF YOU WANT TO FOLLOW-UP,
THAT'S YOUR DECISION.
>> THANK YOU, DR. BIANCHI.
>> ANY OTHER QUESTIONS OR
COMMENTS?
I CAN'T TELL YOU HOW IMPORTANT
AND VALUABLE THIS IS TO ALL OF
US TO HEAR FROM SOMEONE WITH
LIVED EXPERIENCE.
I HOPE, CLAIR, MEMBERS OF YOUR
LABORATORY ARE ALSO LISTENING
BECAUSE IF I WERE WORKING ON
THIS CONDITION, IT'S INCREDIBLY
INSPIRATIONAL TO HEAR DIRECTLY
FROM SOMEONE AND THAT MAKES IT
EVEN MORE IMPORTANT TO FIND A
CURE OR AT LEAST A TREATMENT FOR
THIS CONDITION.
I BELIEVE THAT FINDING THE GENE
THAT CAUSES THE PROBLEM IS HALF
THE BATTLE AND PEOPLE LIKE
DR. LE PICHON AND DR. MOHASSEL
WILL WORK ON THE WAY THE PROTEIN
INTERACTS AND HOW EITHER THE
GENE MUTATION CAN BE AFFECTED OR
ALTERNATE PATHWAYS THAT CAN
PROVIDE A SUBSTITUTE PROTEIN,
FOR EXAMPLE.
>> ABSOLUTELY.
>> THAT'S ONE ASPECT.
THE OTHER IS THE GENETIC ASPECT
AND THAT DOESN'T REQUIRE FUTURE
RESEARCH.
THAT'S AVAILABLE NOW.
SO WITH THAT, I THINK WE'RE
GOING TO ASK YOU AND
DR. MOHASSEL TO SIGN OFF WITH
OUR GREATER APPRECIATION AND
GRATITUDE.
WE REALLY APPRECIATE BOTH OF
YOUR TIME AND IT WAS A BEAUTIFUL
CONNECTION WITH THE STORY OF
DR. LE PICHON'S LAB RESEARCH.
>> THANK YOU SO MUCH.
I APPRECIATE YOU HAVING ME.
>> THANK YOU VERY MUCH.
>> NEXT WE HAVE THE OPPORTUNITY
TO LISTEN TO A PRESENTATION BY A
DIFFERENT REBEKAH RASOOLY ABOUT
THE PROCESS OF SPECIAL COUNCIL
REVIEW.
>> THANK YOU.
SOME HAVE BEEN ON COUNCIL ON
COUNCIL REVIEW.
THIS WAS A PROCESS OR POLICY
STARTED AT NIH ABOUT 10 YEARS
AGO.
WHAT IT MANDATED WAS WHEN A P.I.
WAS RECEIVING LARGE AMOUNTS OF
FUNDING ALREADY, THAT ANY NEWER
APPLICATION BE CONSIDERED BY THE
ADVISORY COUNCIL.
AND INITIALLY THAT THRESHOLD WAS
A MILLION DOLLARS OF DIRECT
COSTS.
THIS YEAR WE RAISED IT TO $2
MILLION OF COSTS.
TO REMIND YOU THE WAY THIS
WORKED OPERATIONALLY IS COUNT
THE RESEARCH PROJECTS GRANTS AND
EXCLUDED TRAINING AWARDS OR
RESOURCE AWARDS AND YOU'VE SEEN
ONE OR TWO OR THREE OR FOUR OF
THESE IN ANY GIVEN COUNCIL NOW.
WE'D LIKE TO TAKE ANOTHER LOOK
AT HOW WE CONSIDER THE
APPLICATIONS FROM VERY WELL
FUNDED INVESTIGATORS.
AND ONE OF THE MOTIVATIONS FOR
DOING THAT IS WORK THAT'S COME
OUT OF NIH LOOKING AT OUR ENTIRE
PORTFOLIO OVER THE LAST SEVERAL
YEARS AND WE DETERMINED THAT
THERE'S A CONCENTRATION OF
RESOURCES WITHIN A RELATIVELY
SMALL PROPORTION OF
INVESTIGATORS SUCH THAT THE TOP
1% OF INVESTIGATORS IN TERMS OF
FUNDING ARE RECEIVING LIKE 10%
OF ALL NIH FUNDING AND IF YOU
LOOK AT THE TOP 10%, THEY'RE
RECEIVING BETWEEN 35% AND 40% OF
ALL NIH FUNDING.
SO IT'S VERY MUCH AN INTENSE
CONCENTRATION.
WHEN YOU LOOK AT THE TOP 10%,
YOU SEE THEY TEND TO BE ON
AVERAGE, MORE MALE, MORE WHITE
AND LIST HISPANIC THAN THE OTHER
POOL OF INVESTIGATORS THAT
THEY'RE BEING COMPARED TO.
THIS IS SOMETHING WE'RE LOOKING
AT AS AN AGENCY AND THERE ARE
DIFFERENT WAYS TO LOOK AT HOW TO
BREAK UP THE CONCENTRATION OF
FUNDING WHICH ALSO LEADS TO A
BIT OF CONSTRAINTING OF RESEARCH
AND RESEARCH AVENUES WHEN YOU
CONCENTRATE SO MUCH FUNDING IN A
SMALL GROUP OF PEOPLE.
WE CAN SAY ANYONE WHO RECEIVES
$1 MILLION IN TOTAL COSTS WOULD
BE A WELL-FUNDED INVESTIGATOR
AND THEIR NEW APPLICATIONS WOULD
HAVE TO GO TO SPECIAL COUNCIL
REVIEW.
THAT'S ONE POSSIBILITY.
ANOTHER POSSIBILITY WOULD BE TO
LOWER THE PAY LINE FOR SUCH
INDIVIDUALS.
IF YOU'RE WELL FUNDED YOUR
APPLICATION WOULD HAVE TO ASSUME
BETTER THAN 10 PERCENTILE, FIFTH
PERCENTILE OR SOMETHING TO BE
CONSIDERED.
WE COULD TAKE STEPS TO REDUCE
THE NUMBER OF APPLICATIONS
COMING FROM WELL-FUNDED
INVESTIGATORS BY SIMPLY
REQUIRING A HIGHER EFFORT LEVEL
ON SUCH APPLICATIONS AND IN THE
END THERE'S ONLY SO MANY GRANT
APPLICATIONS IN WHICH YOU CAN
HAVE 20% EFFORT BEFORE YOU RUN
OUT.
AND SOMETHING ELSE WOULD BE TO
CLOSE THE OTHER LOOPHOLES.
I'LL TALK A LITTLE BIT MORE
ABOUT THAT IN A MOMENT.
HERE'S WHAT OTHER INSTITUTE
COLLEAGUES ARE DOING.
THE THEY HAVE LOOKED AT SAY
WHOLE PROGRAM.
THEY MOVED TO AN INVESTIGATOR
POLICY THAT LOOKED AT NIH
FUNDING NOT -- NINDS HAS ADOPTED
A LOWER PAY LINE AND A THRESHOLD
FOR INVESTIGATORS AND HIGHER
MINIMUM EFFORT AS WELL AS
INCLUDING CHANGING SOME OF THE
RULES SO THAT THEY'RE A
WELL-FUNDED INVESTIGATOR IS ONE
OF SEVERAL INVESTIGATORS ON
APPLICATIONS WE WILL NEVER THE
LESS GET MORE SCRUTINY TO THAT
APPLICATION.
AT THIS MOMENT THEY'D ORDINARILY
BE EXEMPT IF I HAD ONE
WELL-FUNDED INVESTIGATOR AND THE
OTHER INVESTIGATOR IS NOT WELL
FUNDED WE DON'T CONSIDER THAT
FOR SPECIAL COUNCIL REVIEW.
IT'S JUST A REGULAR APPLICATION.
THEY'VE CHANGED THAT RULE.
NIM HAS MADE A LOWER PAY LINE
AND TRYING TO INSIST ON HIGHER
MINIMUM EFFORT.H HAS MADE A LOW
AND TRYING TO INSIST ON HIGHER
MINIMUM EFFORT.
THIS IS WHAT WE'RE PROPOSING AND
FOR YOU TO CONSIDER AND HOPE
WE'LL HAVE A DISCUSSION ABOUT
THIS.
WE'RE PROPOSING THAT A WELL
FUNDED INVESTIGATOR WHO ALREADY
HAS $2 MILLION IN TOTAL COSTS
FROM NIH FUNDING, THAT THEY ONLY
BE EVEN CONSIDERED FOR FUNDING
AND BROUGHT TO COUNCIL FOR
CONSIDERATION IF THEY MEET ONE
OF THE FOLLOWING TWO CRITERIA.
EITHER IT'S AN OUTSTANDING SCORE
OR LESS THAN SIX PERCENTILE, OR
IT REPRESENTS AN NICHD PRIORITY
AREA WE'VE ARTICULATED IN OUR
STRATEGIC PLAN, OR THE
INVESTIGATOR AND THE TEAM THEY
ASSEMBLED HAS THE EFFECT OF
MARKLY AND DISTINCTLY ENHANCING
WORKFORCE DIVERSITY.
SO THE APPLICATION COMES IN WE
REQUIRE A MINIMUM 20% EFFORT IN
THE APPLICATION AND THEY
TRANSITION FROM THEIR
WELL-FUNDED STATUS WITHIN A
YEAR.
FINALLY, CLOSE A MAJOR LOOPHOLE
AND I EXPLAINED IF AN
APPLICATION COMES IN FROM A
WELL-FUNDED INVESTIGATOR AND
PART OF A MULTIPLE INVESTIGATOR
TEAM WE ONLY LOOK AT IT AS A
WELL-FUNDED PROJECT IF ALL THE
INVESTIGATORS ARE IN THAT AND WE
FOUND 95% OF APPLICATIONS ARE
FROM WELL-FUNDED P.I.s WE'RE
PROPOSING TO CLOSE THAT LOOPHOLE
AND IF THEY COME IN WITH AN
APPLICATION AS ONE OF SINGLE
DIGITER THAT IS SEVERAL
NEVERTHELESS PUT IT INTO THE
BUCKET OF WELL-FUNDED
INVESTIGATOR APPLICATIONS.
SO THAT'S OUR PROPOSAL.
MOVING ELIGIBILITY CRITERIA AND
WE INCREASE OUR DEFINITION OF
WELL-FUNDED INVESTIGATORS TO
INCLUDE WHEN THEY ARE ALONG
SEVERAL MULTIPLE P.I.s ON AN
APPLICATION .
THIS IS WHAT WE'RE PROPOSING AND
I'M EAGER TO HEAR YOUR THOUGHTS
ON THIS.
>> THE $2 MILLION IS $2 MILLION
DIRECT PLUS INDIRECT?
>> PLUS INDIRECT.
>> OKAY.
AND I HAVE ANOTHER QUESTION.
I FEEL LIKE OVER THE PAST TWO
PLUS YEARS I'VE BEEN ON THE
COUNCIL WE'VE HAD A FEW OF THESE
AND THEY ALWAYS SEEM TO PASS.
I'VE NEVER SEEN ONE THAT DOESN'T
PASS.
I'M WONDERING WHICH MEANS THE
RULES DON'T MATTER SO MUCH
BECAUSE WE ALWAYS AS A COUNCIL
SEEM TO PASS THEM ANYWAY.
ARE THERE THINGS THAT PROGRAM
ARE NOT BRINGING TO US THAT THEY
HAVE RULED OUT THEY DON'T WANT
TO TRY AND PASS WITH US?
>> YES.
>> I'M WONDERING IF IT'S WISE TO
PROPOSE ALL OF THEM AS ONCE AND
95% AREN'T BEING CONSIDERED TO
COME TO COUNCIL AND IMPLEMENT
EACH OF THE IN A TIME SERIES TO
SEE WHAT AFFECT EACH ONE HAS
VERSUS ALTOGETHER AT THE SAME
TIME.
>> THAT IS ALSO A GOOD
SUGGESTION.
I SHOULD SAY TO YOUR POINT ABOUT
WORKING ON THESE CONSIDERATIONS
THAT ONCE WE GET TO THE OTHER
95%, THAT'S A LOT OF
APPLICATIONS.
>> DR. WONG.
>> YOU SAID 1 MILLION OR $2
MILLION IS THAT TOTAL?
>> WHEN WE DO THE CALCULATION,
AND FORTUNATELY WE DON'T
ACTUALLY DO THE CALCULATION,
IT'S AN ALGORITHM BUILT IN OUR
SYSTEM AND LOOK AT THE SUPPORT
OF THE INVESTIGATOR IN A YEAR.
>> IN A YEAR.
THANK YOU.
AND JUST TO CLARIFY, YOU ARE
SAYING THAT YOU'RE PROPOSING TO
INCLUDE ONLY THE RESEARCH
PROJECT GRANTS OR ALSO THE
RESOURCE GRANTS?
>> ONLY RESEARCH PROJECT.
>> ONLY RESEARCH.
THANK YOU.
>> DR. TITA.
>> THANK YOU FOR THAT
PRESENTATION.
I TOTALLY UNDERSTAND THE NEED TO
DO THIS.
AND GET MORE FUNDED.
HAS THERE BEEN A SYSTEMATIC
REVIEW TO SHOW THE SPECIAL
COUNCIL REVIEW HELPS?
IT MAY BE IT HOLDS A FEW LARGE
GRANT FUNDED DOES IT HELP THE
PROCESS TO GET MORE FUNDING OUT
TO OTHERS?
AND THEN I ALSO WORRY ABOUT
GOING FOR MULTIPLE P.I.
MECHANISM THAT IS SUPPOSED TO
PROMOTE TIM SCIENCE AND SOMEONE
MAY BE WELL FUND ED AND WORKING
TO GET IMPORTANT WORK DONE AND
RAISING THAT BAR AS WELL NOT
JUST LIKE REQUIRING A LOWER
SCORE OR A HIGHER SCORE
DEPENDING ON HOW YOU LOOK AT IT.
DOES IT ULTIMATELY NEGATIVELY
AFFECT THE TEAM SCIENCE?
I WONDER IF THERE'S A WAY TO
OBJECTIVELY EVALUATE THIS TYPE
OF INITIATIVE.
THANK YOU.
>> THANK YOU FOR RAISING THAT
POINT.
SOME OF THE COMMENTS AROUND THE
POTENTIAL UNINTENDED
CONSEQUENCES ON THE MPI SIDE OF
THINGS WHERE ESSENTIALLY THERE'S
A DISINCENTIVE FOR P.I.s TO
ESSENTIALLY CREATE CERTAIN MPIs
OR POTENTIALLY LIMIT WHAT
INVARIABLY HAPPENS IS
COMBINATIONS OF P.I.s WHERE ONE
OR THE P.I. IS THE HUB P.I. AND
THEY MAY BE WELL ESTABLISHED AND
PROVIDE RESOURCES FROM OTHER
P.I.s THAT BRING EXPERTISE OR
RESOURCES.
IN SOME RESPECT YOU LIMIT THE
POTENTIAL FOR THE OTHER P.I.s
WHO MAY NOT BE ABLE TO SORT OF
OBTAIN A GRANT INDEPENDENTLY OR
POTENTIALLY DESIGNATING REDUCING
THE OWNERSHIP INSTEAD OF MPI
THEY'LL BE CO-INVESTIGATORS OR
SOMETHING ALONG THOSE LINES OF
P.I.s YOU WANT TO HAVE EFFORT
ADDRESSED AND THE OTHER PART
THAT SEEMS IS ALSO I'M WONDERING
IS CONSIDERED IS THERE'S A WIDE
RANGE OF INDIRECT COSTS ACROSS
DESTINATIONS.
YOU MAY INADVERTENTLY HAVE
AFFECTS AT SOME INSTITUTIONS
VERSES OTHERS IN THE SETTING.
RECOGNIZING THERE'S PROBABLY
SOME CORRELATION BETWEEN
INSTITUTIONS THAT HAVE HIGH FNA
RATES AND FUNDED INVESTIGATORS.
I WONDER IF THAT MIGHT BE ALSO
UNINTENDED DISPARITY TO THE
P.I.s WHO THEMSELVES DON'T GET
TO CONTROL NECESSARILY THE
INDIRECT RATES OF THEIR
INSTITUTIONS.
AND THIRD, THE QUESTION AROUND
THE TRANSITIONING -- I WONDER IF
THERE'S A RISK WITH THE SHORT
TIME LINE ON THE PROGRAMS THAT
THEY'RE TRANSITIONING FROM.
MEANING WHAT ARE CONTRIBUTIONS
AROUND THE TIME LINES AND
AFFECTS IT MAY HAVE ON PROGRAMS
THEY'RE FUNDED ON WHEN THERE'S
ONLY ONE YEAR?
SCIENCE TYPICALLY TAKES A LONGER
TIME COURSE THAN A YEAR.
I WONDER IF IT MIGHT AMPUTATE
SOME PROGRAMS PREMATURELY FOR
INVESTIGATORS.
THOSE ARE JUST SOME THOUGHTS OR
QUESTIONS.
>> THESE ARE EXCELLENT POINTS.
I WANT TO SAY ONE THING.
WHEN WE GET A MULTIPLE P.I.
APPLICATION THE ALGORITHM
APPORTIONS THE FUNDS.
IF IT'S A $2 MILLION GRANT WITH
THREE P.I.s, EACH ONE GETS
CHARGED $2 MILLION EACH ONE GETS
CHARGED ABOUT A THIRD OF THAT
AND IF IT'S A GRANT WITH
MULTIPLE PROJECTS, THEN IT WOULD
BE A PROPORTION.
I DID WANT TO MAKE THAT ONE
SMALL POINT.
>> IT'S HELPFUL.
THANKS.
>> DR. FAIR.
>> YEAH, I'VE BEEN THINKING
ABOUT THIS PROBLEM FOR A WHILE.
SOMETIMES THE WAY THE SYSTEM IS
SET UP MAKES IT HARD FOR SOME
INVESTIGATORS AND THERE'S A CUL
TIRE IN OUR STUDY SECTIONS --
CULTURE IN OUR STUDY SECTIONS
THAT LOVE SENIOR INVESTIGATORS
SO IT HELPS ME GET GRANTS FOR
YOUNG INVESTIGATORS BECAUSE
THAT'S THE CULTURE AND REALITY.
I LOVE THE IDEA BUT WHAT'S THE
CONSEQUENCE IN THE CURRENT
CULTURE.
I LIKE THE CARROTS AND WHETHER
YOU CAN QUANTIFY THIS WHEN YOU
COME TO COUNCIL.
YOU TALK ABOUT LIKE IF 50% OF
STAFF IS UNDER REPRESENTED YOU
GET TREATED LIKE EVERYBODY ELSE.
THIS IS A POTENTIAL OPPORTUNITY
TO PUSH WHAT WE TALK ABOUT
BECAUSE THERE'S A HUGE INCENTIVE
FOR THAT.
I DON'T KNOW WHAT'S POSSIBLE BUT
THAT PART OF THE PLAN IS WHAT I
LATCHED ON TO RIGHT AWAY BECAUSE
IT HAS SIGNIFICANT POWER TO KILL
A MULTIPLE OF BIRDS WITH ONE
STONE IF YOU DO IT RIGHT.
>> I HEARD IN PAST COUNCILS THE
COMMENT ABOUT THOSE WHO HAVE
MORE, GET MORE AND IT BECOMES
PERPETUATING THAT WAY AND WAS
THINKING ABOUT THE IDEA OF DEI
AND HAVING A METRIC IN THERE SO
A PERSON CAN ACTIVELY
DEMONSTRATE YOU ARE DIVERSE
TYING YOUR TEAM.
-- DIVERSIFYING TEAM AND WITH
THE 3% EFFORT AND MAYBE YOU SAID
THIS OR MAYBE I MISSED ON THIS,
ARE WE TALKING ABOUT AN
INVESTIGATOR WHERE THERE'S SCANT
5% OR LESS OF VERY LOW EFFORT ON
ACROSS AWARDS AND SO ONE WONDERS
GIVEN THE IDEA, THE SPARK OF
WHAT THE RESEARCH IS BUT WHAT
ADDITIONALLY IS THE INVESTIGATOR
IN ADDITION TO THEIR NAME AND
REPUTATION, GIVING TO THE ACTUAL
WORK ARE WE TALKING ABOUT
GETTING UP TO 5% BECAUSE IT'S SO
LOW OR UP TO 10%?
I'M WONDERING ALOUD ABOUT THE
PERCENT EFFORT INCREASE WHERE
WE'RE STARTING FROM ON GETTING
IT UP TO SOMETHING.
WHEN THE GRANT IS AWARDED
THEY'LL HAVE TO ESTABLISH THE
PERCENT EFFORT ON THE NEW GRANT.
>> I WOULD IMAGINE YOU'RE NOT
SEEING ANYTHING CLOSE TO THAT AT
ALL.
THE 20% --
>> IT'S ACROSS THE SPECTRUM.
>> AND THE IDEA OF JUSTIFYING
WHAT YOU'LL DO IN RELATION TO
THE CONDUCTS ABOUT THAT SCIENCE,
THAT IN FACT REPRESENTS 20%
EFFORT FROM YOU KNOWING THAT
THEY'RE WORKING WITH PRETTY
LARGE TEAMS, PROBABLY.
I'M THINKING ALOUD HERE WITH
THAT.
THOSE ARE SOME OF MY INITIAL
THOUGHTS.
>> DR. WONG.
>> THANK YOU.
I WANTED TO FOLLOW-UP ON THE
COMMENT BY DR. LANG AND RESNIK
THAT SOME OF THE PROPOSALS CAN
BE QUANTIFIED AND SEE WHAT WOULD
BE THE EFFECT.
WHAT WOULD BE THE IMPACT IF WE
IMPOSE THIS, THIS WOULD HAPPEN.
IF WE IMPOSE THAT, THAT WOULD
HAPPEN.
SO IN TRYING TO ASSESS THE
POSSIBLE IMPACT OF THESE CHANGES
IN PARTICULAR WHETHER THERE'S
SOME PROPOSAL THAT IS CONSIDERED
VALUABLE BY YOU ALL IN THE
PROGRAM WOULD BE RESULTING IN
NON-FUNDING, FOR EXAMPLE.
THAT IS WHAT IS OF CONCERN TO US
WHETHER THE WORD AMPUTATE WAS
USED.
PARTICULARLY GOOD SCIENCE WOULD
BE MISSED SOME NEW DISCOVERY.
SOMETHING THAT A SENIOR
PROFESSOR WITH A LARGE PROGRAM
IS ADVANCING AND LEADING LIKE
YOU WERE SAYING.
THIS IS AWARDED BY THE STUDY
SECTIONS AND THE SENIOR FACULTY
LEADING THE EFFORT AND WE KNOW
THIS IS GOING TO BE DONE.
AND THEY REWARD THAT A LOT.
THE EXPERIENCED INVESTIGATOR WHO
HAS CONDUCTED THIS WORK WILL BE
LEADING THE EFFORTS.
THIS IS A VERY GOOD PROPOSAL AND
WE KNOW THAT THIS IS GOING TO BE
DONE.
I'M SUPPORTING THE POINT THAT
PERHAPS WE PROBABLY WOULD NEED
TO CHANGE THE CULTURE OF THE
STUDY SECTION AND I'M SORRY, I
HAVE TWO MORE POINTS.
ONE HAS TO DO WITH THE PROPOSAL
TO EXCLUDE CERTAIN AWARDS.
AND SO I'M GOING SPEAK NOW AS A
P.I. AND HAS AN RO1 IN
POPULATION STUDIES WE DO WORK
WITH RO1s NOT U OR OTHERS IN
COLLECTING LARGE AMOUNTS OF DATA
THAT ARE THEN PUT UP FOR PUBLIC
VIEWS IN POPULATION STUDIES AND
HAVE NATIONAL SAMPLES AND
LONGITUDINAL STUDIES AND THOSE
ARE VERY EXPENSIVE GRANTS.
THEY'RE OURS.
WE HAVE TO CONDUCT THE STUDIES
AND AGAIN, THERE ARE RESOURCES
BECAUSE THE DATA'S PUBLIC.
IT'S NOT FOR OUR LAB OR OUR
UNIVERSITY, IT'S FOR THE PUBLIC.
FOR US WE SET IT UP FOR PUBLIC
USE AND SHARE THE DATA BUT IT'S
AN "R".
PERHAPS THE KIND OF GRANT OR
AWARD IT IS SHOULD BE CONSIDERED
IN THERE SO NOT ALL R01s ARE THE
SAME OR HAVE THE SAME IMPACT.
THAT'S WHY THE PRICE -- THE COST
GETS HIGHER.
THE OTHER THOUGHT I HAVE, SORRY
FOR TAKING SO LONG, I WAS
THINKING IN THE PUBLICATION
RECORDS WE HAVE WE TEND TO BE
FIRST AUTHORS AND THEN SENIOR
AUTHORS.
THE CULTURE OF PERHAPS IN THE
STUDY SECTIONS WOULD HAVE TO
CHANGE THAT I COULD BE THE
SENIOR INVESTIGATOR WITHOUT
BEING THE P.I.
AND THEN THE P.I.s THAT GO IN
FRONT ARE THE ONES I'M
SUPPORTING AND I'M SAYING THAT
I'M GOING TO BE FULLY INVESTED
IN THIS RESEARCH PROJECT BUT I'M
GOING TO BE THE SENIOR
INVESTIGATOR AS THE LAST SO SOME
THOUGHT HOW THE STUDY SECTION
WOULD CONSIDER, OKAY, SHE'S
GOING TO BE IN THERE AND GOING
TO BE FULLY SUPPORTED BUT SHE'S
NOT MENTIONING HER NAME AS P.I.
BECAUSE SHE'S PUSHING THE OTHERS
AND BEING THE FIRST AUTHORS, THE
EQUIVALENT OF THAT.
>> EXCELLENT POINT.
>> WE HAVE A NICE DISCUSSION
HERE BUT I THINK WE SHOULD
CONSIDER THE TEAMS.
PARTICULARLY FOR THESE MULTIPLE
P.I. GRANTS BECAUSE THERE'S
EVIDENCE THAT OPTIMIZING THE
TEAM WOULD LEAD TO MORE IMPACT
OF THE WORK.
NORMALLY WE HAVE A STATEMENT
ABOUT COLLABORATION AND THAT
COULD INCLUDE DIVERSITY.
THERE ARE MANY PHASES TO
OPTIMIZING THE TEAM LEVEL CAREER
DEVELOP AND ALL THAT AND THESE
ARE PEOPLE WITH LARGE SALARIES
AND IF YOU DO THAT IT'S GOING TO
BE HARD SOMETIMES HARD TO GET
THE WORK DONE WITHIN THE BUDGET.
SOMETIMES JUST INCREASING THE
EFFORT -- SOMETIMES I NEED TO
DECREASE YOUR EFFORT TO HIRE THE
OTHER PEOPLE YOU NEED ON YOUR
TEAM.
AND THE OTHER ASPECT IS LOOKING
AT THE STUDY SECTION.
WHY THE STUDY SECTIONS ARE NOT
TAKING THIS INTO MORE
CONSIDERATION.
THIS ASPECT.
I'M SURPRISED TO HEAR THE
DISCOUNT OF THAT -- WE HAVEN'T
HAD ANY REJECTIONS IN THE PAST
FOR THESE TYPE OF APPLICATIONS
HERE.
WE GET THE MINORITY RIGHT OF THE
APPLICATION AND I'D LIKE TO KNOW
MORE HOW MANY DON'T MAKE IT
HERE.
WHAT ARE THE PROPORTION OF
APPLICANTS IN THIS CONDITION
THAT WE GET HERE?
THEY'RE BEING ACCEPTED.
THANK YOU.
>> THANK YOU.
>> I JUST WANTED TO COME BACK TO
WHAT DR. FAIR SAID ABOUT
CULTURE.
BECAUSE I THINK CULTURE ON STUDY
SECTION IS A HUGE PART OF THIS.
BECAUSE THERE'S SO MUCH CREDIT
GIVEN TO SENIOR INVESTIGATORS
THAT I THINK WITHOUT ADDRESSING
THE CULTURE THAT THIS IS GOING
TO BE A MAJOR CHALLENGE AND I
ALSO WANTED TO ALSO AGREE WITH
THE COMMENTS ABOUT EFFORT.
I APPRECIATE WHAT THE GOAL IS
AND I THINK IT'S REALLY
IMPORTANT BUT A LOT OF TIMES
PEOPLE WHO HAVE A LOW EFFORT ARE
REALLY TRYING TO SAY SAVE BUDGET
TO BE ABLE TO PAY FOR STAFF AND
TRAINEES AND LEARNERS.
I THINK THAT COULD POTENTIALLY
CRIPPLE SOME PROGRAMS BECAUSE
THEY HAVE OTHER SOURCES FOR
THEIR SALARY WHICH ALLOWS THEM
TO DO THAT AND IT ACTUALLY MAKES
SENSE TO USE THE FUNDS WHERE THE
FUNDS ARE NEED ED I ALSO WANT T
GIVE A NOD TOWARDS DIVERSITY
CHALLENGE AND WHEN YOU SIT IN
STUDY SECTION AND THE QUESTION
AROUND DIVERSITY IS RAISED IS
ALWAYS WELL, THEY'LL DO IT.
THEY'LL GET IT DONE.
FROM MY PERSPECTIVE IT OFTEN
COMES BACK AND DOESN'T GET DONE
AND HAVING SOMETHING MORE
CONCRETE WOULD BE A GOOD THING
AND DIVERSITY IS MANY THINGS.
IT'S DISABILITIES, IT'S RACE,
IT'S SEX AND DEPENDING ON THE
NEEDS ON THAT KIND OF SYSTEM, WE
HAVE SOME STATEMENTS WITH HARD
NUMBERS WOULD GO A LONG WAY.
>> I DON'T THANK WE'RE GOING TO
CHANGE THE STUDY SECTION CULTURE
ON COUNCIL.
I DON'T THINK IT WILL HAPPEN
QUICKLY.
SECOND POINT IS I THINK IT WOULD
BE GOOD FOR COUNCIL TO REVIEW
ANY OF THESE APPLICATIONS THAT
HAVE A GRANT ABOVE A MILLION
DOLLARS WHEN THEY COME IN AND
THOUGH THE COMMENTS ARE TRUE
FROM CATHERINE AND PEPE WE
HAVEN'T NOT REVERSED ANY OF THEM
THAT'S BECAUSE THEY WERE ALL AT
THE LIMIT AND THEY WOULD TURN
OVER IN THE NEXT YEAR.
I THINK THEY'VE ALL BEEN WITHIN
GOOD PARAMETERS TO FUND THEM.
AND I DON'T HAVE ANY PROBLEM
WITH PASSING THEM THROUGH BUT I
THINK A GOOD DISCUSSION WOULD BE
HELPFUL AND I THINK ELEVATING
SOME OF THESE TOPICS THAT WERE
BROUGHT UP AS A MEANS OF
REVIEWING THE DIVERSITY ASPECTS,
WHETHER THIS TEAM IS A BIGGER
TEAM IS NEEDED FOR THE KIND OF
RESEARCH THEY DO, THIS IS THE
KIND OF THING THAT COULD BE
DISCUSSED AT COUNCIL FIRST FROM
THE PROGRAM PEOPLE SAYING THIS
IS WHY WE THINK IT'S GOOD OR NOT
GOOD, WHATEVER IT COMES UP IT
COULD BE DISCUSSED AT COUNCIL.
AND I WON'T HAVE TO DO IT ANYWAY
BECAUSE THIS IS MY LAST MEETING.
>> I'M LOOKING AT THE TIME AND
WE'RE GETTING A LITTLE BIT
BEHIND TIME.
DR. MALDONADO.
>> I WAS HAVING TROUBLE AND
LEAVING MY VIDEO OFF.
I AGREE WITH COMMENTS AND WHEN I
INTRODUCED MYSELF I'M ALSO
SENIOR ASSOCIATE GENE FOR
DIVERSITY AT THE STANFORD SCHOOL
OF MEDICINE AND I THINK WE'RE
ALL AWARE OF THE DATA AROUND
JUNIOR INVESTIGATORS AND THE
TIME TO FIRST START RO1.
IT'S INCREDIBLY EXTENDED.
THE CURRENT AGE AS A MEAN OF 45
YEARS OF AGE.
WE ARE -- I WOULD LIKE TO SEE
MORE MEANS TESTING OF THEE
OPTIONS TO SEE I'M AN
EPIDEMIOLOGIST AND SEE WHO YOU
WOULD INCLUDE AND EXCLUDE.
I WANT TO MAKE SURE WE'RE
REWARDING THE SCHOLARSHIP BUT
ALSO INCLUDING AS MANY OF THE
BOTTOM 50% DISTRIBUTING THAT
BETTER.
MAYBE MEANS TESTING AND I'M SURE
YOU'VE ALREADY DONE SOME OF THIS
BUT AS A FIRST PASS I DON'T KNOW
EXACTLY HOW MUCH YOU WOULD
CHANGE THE PROFILE AND THIS IS A
MAJOR POLICY SHIFT.
I THINK A LITTLE BIT MORE FOR
US.
I DON'T KNOW WHEN YOU'RE GOING
TO IMPLEMENT THIS BUT I WOULD
SAY GIVING THIS A LITTLE BIT
MORE OF AN UNDERSTANDING OF WHO
WOULD BE INCLUDED BY SOME OF THE
MEASURES AND WHAT THE
SENSITIVITY ANALYSIS AROUND THAT
AND DIVERSIFYING THE WORKPLACE
APPLICATIONS HOW THAT WOULD
IMPACT THIS PARTICULAR GROUP.
THE SECOND AREA I THINK THAT'S
REALLY IMPORTANT, I COMMISSIONED
A TASK FORCE ON IMPACT OF
COVID-19 ON WOMEN FACULTY AT
STANFORD.
WE PUT TOGETHER A TOOL KIT OF
IMMEDIATE ACTIONS WE COULD TAKE
AS DEPARTMENT CHAIRS AND OTHER
LEADERS TO SUPPORT YOUNG WOMEN
INVESTIGATORS BUT WE REALIZE
THIS IS OBVIOUSLY NOT A GENDERED
ISSUE.
IT TENDS TO HAVE A GENDER IMPACT
DISPARATE BUT IT'S AN IMPACT
OVER ALL ON JUNIOR FACULTY.
I DO THINK THAT
[AUDIO DIGITIZING]
AND IT'S HARD TO EXCLUDE SENIOR
FACULTY WHEN THEY ARE PROBABLY
THEIR C.V.s AND TRACK RECORDS
WILL OVERWHELM WHAT'S GOING ON
WITH THE JUNIOR FACULTY AND IN
THE LAST TWO YEARS THEY'VE BEEN
ABLE TO EXPAND THEIR TRAJECTORY
AS WELL AS THE SENIOR PEOPLE
HAVE DONE.
I THINK A DIFFERENT MECHANISM
FOR FOCUSSING ON JUNIOR FACULTY
APPLICATIONS WOULD BE BEYOND THE
FIRST AWARD AND MAYBE INCREASING
ACCESS TO FIRST AWARDS WOULD BE
HELPFUL AND THIRD --
[AUDIO DIGITIZING]
>> WE'RE HAVING TROUBLE.
>> SORRY ABOUT THE CONNECTION.
SILICON VALLEY ISN'T WHAT IT'S
SUPPOSED TO BE.
I CAN TELL YOU THAT I STILL
THINK THE CULTURE NEEDS MAJOR
REVAMPING.
IT'S STILL A QUAGMIRE IN I HAVE
VIEW.
I THINK THE PANDEMIC HAS ONLY
MADE IT WORSE.
I DON'T KNOW HOW TO ADDRESS THAT
CLEARLY.
I WOULD SUGGEST THAT IS A MAJOR
AREA AND DOING THE WORK-AROUNDS
AROUND THE STUDY SECTION IS
HELPFUL BUT IT'S NOT GOING TO BE
A FUNDAMENTAL CHANGE UNTIL WE
CHANGE THE CULTURE OF THE STUDY
SECTIONS.
>> DO YOU WANT TO GIVE THE LAST
WORD TO DR. FAIR?
>> I DO WANT TO ADDRESS SOME
COMMENTS BUT I WANT TO HEAR FROM
DR. FAIR.
>> I'LL BE QUICK.
I WANT TO ADDRESS THE COMMENT
THAT WON'T CHANGE THE STUDY
SECTION BUT AND THIS IS A
THOUGHTFUL POLICY.
WE'RE TRYING TO THINK CREATIVELY
TO IMPLEMENT IT AND WE HAVE
THOUGHTFUL DIRECTORS AND
PROGRAMS AND HOPEFULLY YOU CAN
BRING THE MESSAGE BECAUSE IT'S
SUCH A BIG PROBLEM FOR DOING THE
THING YOU WANT.
I WANT TO PUT THAT OUT THERE.
>> THANK YOU.
IF I CAN MAKE A DIRECT COMMENT
AND THEN WE'LL MOVE ON.
THERE'S THREE LEVELS OF PLACES
TO INTERACT.
EVERYONE IS AWARE OF THE ISSUES
WITH THE COUNCIL STUDY SECTIONS.
AND NONI BURNS HAS IMPLEMENTED
AN IMPLICIT BIAS TRAINING COURSE
SO FOR THOSE REVIEWERS, YOU'RE
EITHER GOING TO TAKE IT SOON OR
YOU HAVE TAKEN IT BUT FROM WHAT
HE HEARD FROM HER, IT'S BEEN
VERY WELL REVIEWED AND
APPRECIATED.
PEOPLE HAVE COMMENTED THEY WERE
NOT AWARE OF THEIR BIASES WHICH
INCLUDES BIASES TOWARDS SENIOR
INVESTIGATORS WHO AS REBECCA AND
SHAWNA TEND TO BE WHITE AND MALE
AND IT AFFECTS SOME OF THE
REVIEWERS FOR THE NICHD INTERNAL
STUDY SECTIONS, WE NEED TO
IMPLEMENT THAT SAME KIND OF
TRAINING INTO OUR OWN STUDY
SECTIONS.
THAT'S NUMBER ONE.
NUMBER TWO, YOU HEARD OF WHAT
WE'RE DOING INSTITUTE WIDE BUT
NUMBER THREE AT THE BEGINNING
WHEN YOU TALKED ABOUT DR. TABAK
AND DR. TABAK MOVING FROM ACTING
DIRECTOR TO MORE AND HE'S
EXTREMELY AWARE OF THIS AND ITS
CONNECTION TO YOUNG
INVESTIGATORS AND INVESTIGATORS
FROM UNDER REPRESENTED
MINORITIES.
THE DATA SHOWS THEY'RE
OVERWHELMINGLY WHITE AND MALE
AND IT CLOSE THE OPPORTUNITIES
TO DIVERSE IF I THE WORKFORCE.
IN THE OFFICE OF THE DIRECTOR
THEY'RE DOING QUITE A LOT OF
DATA MODELLING TO SEE WHAT THEY
CAN DO AT A HIGH LEVEL AND I
WANT TO LEAVE THE MESSAGE THIS
IS A VERY CRUCIAL AREA WITHIN
THE INSTITUTES AND THE OFFICE OF
THE DIRECTOR OF NIH.
HOPEFULLY WE'LL SEE MAJOR
CHANGES.
>> THANK YOU FOR THE
CONVERSATION.
WE ANTICIPATED A LOT OF INPUT
AND WE'RE GRATEFUL.
IT'S NOT A DONE DEAL.
IT WILL SHAPE OUR POLICIES IN
LIGHT OF YOUR COMMENTS AND IN
LIGHT OF WHAT'S HAPPENING ACROSS
NIH.
>> READY FOR THE NEXT SESSION?
>> WE'RE READY FOR THE NEXT
SECTION.
>> THIS IS -- AND FIRST WE THANK
YOU AND NOTE YOUR COMMENTS IN
THE CHAT BOX AND HOPE WE'LL BE
CAPTURING THEM AND RECORDING
THEM FOR OUR INFORMATION.
THE NEXT SECTION A BITTER SWEET
SECTION BECAUSE WE'RE VERY SAD
WE HAVE QUITE A NUMBER, SIX
PEOPLE RETIRING THIS YEAR AFTER
THEIR DEDICATED SERVICE ON THIS
ADVISORY COUNCIL.
I'M GOING CALL ON PEOPLE IT'S
NOT AN ALPHABETICAL ORDER SO
YOU'LL HAVE TO BEAR WITH ME.
THE FIRST IS DR. MICHELLE
CAGGANA YOU CAN SAY WHATEVER YOU
WANT ON WHAT THE EXPERIENCE WAS
LIKE AND ANY SESSIONS YOU HAVE
FOR US ANYTHING YOU LEARNED.
THE FLOOR IS YOURS.
>> THANK YOU.
I LEARNED A LOT AND I GET TO GO
FIRST SO WE'LL SEE HOW IT GOES.
TIME SURE FLIES.
I WAS APPROACHED IN 2017 AND
ASKED IF I I WANTED TO BE ON
COUNCIL AND I SAID OKAY SO I
SUBMITTED THE MATERIALS I NEEDED
AND WAITED A LONG TIME AND
FINALLY GOT A LETTER SHOWING UP
FROM SECRETARY AZAR THAT WAS
INVITING ME TO JOIN.
I WANTED TO THANK HER FOR ASKING
ME TO APPLY AND GIVING ME THIS
EXPERIENCE AND FOR BEING MY
BUDDY OVER THE PAST FEW YEARS.
ALSO THANKS TO THE NICHD STAFF
AND FOR ANSWERING ALL MY
QUESTIONS.
I WANTED TO THANK YOU,
DR. BIANCHI AND SERNICHE.
AS A NEWBORN SCREENING PERSON
THE HEALTH OF WOMEN AND CHILDREN
ARE HIGH ON MY LIST.
IT'S ALLOWED ME TO WITNESS NEW
INITIATIVES.
I WAS NEAT SEEING THE DIFFERENT
VOICES OF PARTICIPANTS AND HOW
IT EVOLVED OVER TIME TO HAVE
CONCEPTS RELATED TO THE
ENJOYABLE AND EDUCATIONAL
SESSIONS.
ALSO ALL THE GREAT WORK DURING
THE PANDEMIC AND THE AFTER MATH
AND I WAS PRIVILEGED TO SEE THE
UNVEILING OF THE STRATEGIC PLAN
WHICH CONTINUES TO BE THE FOCUS
OF A LOT OF THE WORK WE DO ON
COUNCIL.
I HOPE WE CAN MEET IN PERSON AND
I WAS GOING SAY I'M GRADUATING.
AT LEAST WE GOT TO MEET AS A
GROUP A FEW TIMES BEFORE THE
PANDEMIC HIT.
I'M ALWAYS IMPRESSED BY THE
EXPERTISE AND DEDICATION OF THIS
GROUP.
I HOPE I CONTRIBUTED TO HELPING
THE INSTITUTE'S INITIATIVES AND
I'M LEAVING THE GROUP IN REALLY
GOOD HANDS AND NEW PEOPLE AND MY
TEAM IS ROTATING OFF SO I WANT
TO WISH EVERYONE THE BEST OF
LUCK AND GOOD HEALTH.
THANKS.
>> THANK YOU SO MUCH.
>> WHY'S IT TAKE SO LONG.
WE QUERY INTERESTS AND SOME
PEOPLE HAVE TO BE CLEARED BY THE
DEPARTMENT OF HEALTH AND HUMAN
SERVICES AND SOME PEOPLE GET
REJECTED.
THERE'S ALL KINDS OF THINGS
TAKEN CONSIDERATION.
IT'S NOT A COMMENT ON YOUR
SCHOLARSHIP OR CAREER.
OFTEN IT'S CONFLICT OF INTEREST
OR BALANCING GEOGRAPHIC OR
DIVERSITY NEEDS AND SOMETIMES
POLITICAL AS WELL THAT'S BEYOND
OUR CONTROL.
>> NEXT UP IS DR. MATZUK.
>> MICHELLE SAID A FEW THINGS I
MAY REPEAT.
I'D LIKE TO THANK PAST COUNCIL.
THIS IS AN AMAZINGLY DIVERSE
GROUP AND I'VE APPRECIATED
DIFFERENT PERSPECTIVES THAT
PEOPLE HAVE GIVEN ESPECIALLY
OVER THE LAST 35, 40 MINUTES.
I APPRECIATE THE DIFFERENT
PERSPECTIVES.
I'D ALSO LIKE TO SAY THAT NICHD
IF YOU DIDN'T NOTICE IT BEING ON
COUNCIL, WE HAVE AN AMAZING
BRANCH CHIEF PROGRAM OFFICERS,
SCIENTIFIC REVIEW OFFICERS AND
WE SHOULD REALLY APPRECIATE HOW
THEY GO OUT OF THEIR WAY TO HELP
ALL OF US.
MY WIFE HAS BEEN FUNDED TO HELP
OTHER NIH INSTITUTES AND THERE'S
NO COMPARISON BETWEEN NICHD AND
OTHER INSTITUTES.
IN PARTICULAR I WANTED TO THANK
A FEW OF THE PROGRAM OFFICERS
WITH WHOM I'VE INTERACTED
INCLUDING DR. CHANG AND STEPHEN
CALPIN AND OTHERS AND THERE'S
BEEN WARM INDIVIDUALS THAT HELP
ME AS AN INVESTIGATOR AND WHEN
WE COULD MEET IT WAS FANTASTIC
TO SPEAK WITH THEM IN PERSON.
I SEE PEOPLE ARE GOING TO MEET
AGAIN IN JANUARY.
I WISH THIS WAS THE TIME OF YEAR
WE COULD BE MEETING IN D.C. AND
WISH WE COULD HAVE MET IN PERSON
BECAUSE THE PERSONAL
INTERACTIONS HAVE BEEN TO ME.
ONE OF THE PERSONAL
INTERACTIONS, I'VE KNOWN LOU
DAPALO ABOUT 30 YEARS OR ONE OF
THE LAST TIMES AT THE COUNCIL
MEETING WE HAD LUNCH TOGETHER
AND CAUGHT UP ON DIFFERENT
THINGS.
ALSO LISA HALVERSON ANOTHER
FORMER BRANCH CHIEF A FANTASTIC
INDIVIDUAL.
GREAT PEOPLE SOMETIMES MOVE ON
TO OTHER GREAT OPPORTUNITIES.
AND I WANT TO THANK MY COUNCIL
BUDDY AND MULTI-BRANCH CHIEF
DR. JOHNSTON AND NOW HE'S THREE
BRANCH CHIEFS AND I REALLY
APPRECIATE HIS LEADERSHIP,
GUIDANCE AND DISCUSSIONS PRIOR
TO COUNCIL AND TYPICALLY WE'D
REACH OUT AND BE ON A ZOOM CALL
TALKING ABOUT MY ASSIGNMENTS AND
IF THERE WERE ISSUES OR NEEDED
ANYTHING ELSE AND ANOTHER
INDIVIDUAL WOULD MEET WITH US.
I SPOKE ABOUT MEETINGS BEFORE
THE PANDEMIC.
DAN AND I USED TO GET TOGETHER
FOR DINNERS BEFORE HAND.
ALONG WITH DAN IS STEWART MOSS.
SOME OF THE NEW INDIVIDUALS MAY
NOT KNOW HIM AND MAYBE OTHERS
DON'T KNOW HIM BUT HE'S A GREAT
GUY AND I KNEW HIM IN THE
ACADEMIC ARENA AND HAVE KNOWN
HIM WITH NIH AND WITH STEWART
DYING DURING THE PANDEMIC I MISS
THOSE INTERACTIONS ONE OF THE
LAST DINNERS WERE STEWART AND
LINDSEY AND I AND IT WAS NICE TO
FREELY TALK ABOUT THINGS AND GET
TO KNOW PEOPLE BETTER.
THOSE THINGS I'LL REMEMBER
FOREVER FOR ME AND I CAN'T SAY
ENOUGH ABOUT YOU, DIA DIANA.
IT'S OBVIOUS FROM ALL THE
MEETINGS WE'VE HAD EVENBURY MOTE
YOU AND GAVE A PRESENTATION AT
NIH YOU REALLY HAVE DONE A
FANTASTIC JOB.
YOU'VE SURROUNDED YOURSELF WITH
DIVERSE GREAT PEOPLE AND I
REALLY FEEL THAT NICHD'S IN
GREAT HANDS WITH YOU AT THE HELM
AND ALL OF NIH AND THE FUTURE
DIRECTOR SHOULD KNOW THE ROLE
YOU'VE BEEN PLAYING AND HOW WELL
A JOB YOU'VE BEEN DOING.
>> THANK YOU SO MUCH.
IT'S BEEN GREAT HAVING YOU ON
OUR ADVISORY COUNCIL AND
PARTICULARLY THANK YOU FOR
GIVING SHUT OUTS TO SO MANY OF
OUR STAFF MEMBERS AND NICE HAVE
YOU THE COMPARATOR.
YOU KNOW THROUGH YOUR WIFE'S
WORK HOW DIFFERENT AND SPECIAL
OUR STAFF ARE.
THANK YOU.
>> THANK YOU, MY NAME IS REBEKAH
WONG AND THE NAME IS MISLEADING,
I WAS BORN AND TRAINED IN MEXICO
AND TRAINED IN THE U.S. IN Ph.D.
AND POPULATION ECONOMICS AND SO
HAVING SAID THAT, I'D LIKE TO
REFLECT THREE POINTS.
FIRST, I WAS GIVEN THE PRIVILEGE
LIKE MANY OF US HAVE SAID TO
SERVE ON THIS ADVISORY COMMITTEE
KIND OF NOT EXPECTING THE USUAL
MEETING.
YOU WANT ME TO SERVE ON THIS
BODY AND SO IT WAS A VERY MUCH
AN HONOR TO SERVE AT THE ONLY
SOCIAL SCIENTIST IN THIS GROUP
IN MY FIRST YEAR AND IT WAS LED
BY DR. BIANCHI AND A WONDERFUL
GROUP OF PEOPLE IN COUNCIL AND
OUTSIDE OF COUNCIL.
THE CROSS CUTTING THEMES OF
HEALTH DISPARITIES WAS ALREADY
THERE AND I WAS PLEASANTLY
SURPRISED.
I'LL REFER TO THIS THEME AGAIN.
AND MY SECOND POINT LIKE WE'VE
BEEN SAYING THE SERVICE WAS
MOSTLY VIRTUAL DUE TO THE
PRESIDENT-ELECT PANDEMIC BUT THE
OTHER SIDE OF THE COIN IS THAT
WE WERE ABLE TO SEE HOW THE NIH
REACTS TO A GLOBAL EMERGENCY AND
NIH MISSED NO BEAT.
THE EMERGENCY PROGRAMS WERE
QUICKLY LAUNCHED, RESEARCHERS
RESPONDED TO THE EMERGENCY CALL.
WHAT AN IMPRESSIVE DISPLAY.
WE WERE ALL CONSCIOUS OF THE
DEVASTATION BUT UNDERSTAND THE
UNIQUE ROLE OF SCIENTIST AND
EDUCATORS AND WE HAD TO KEEP
GOING.
WE HAD TO TRY HARDER AND COULD
SEE IT FROM THE INSIDE OF NIH.
THANK YOU, DR. BIANCHI AND ALL
YOUR STAFF, LIKE YOU MENTIONED
TODAY, YOU'RE AWARE OF THE
ENORMITY OF THE EFFORTS.
THAT WAS WONDERFUL TO SEE FROM
THE INSIGHT.
AND MY THIRD AND LAST POINT,
AFTER THE PANDEMIC, I HAD THE
PRIVILEGE TO SERVE IN A WORKING
GROUP PUT TOGETHER BY THE
COUNCIL OF COUNCILS.
YOU GAVE ME THE OPPORTUNITY AND
NOMINATED ME IN RESPONSE TO A
CONGRESSIONAL MANDATE TO
INTEGRATE THE BEHAVIORAL AND
SOCIAL APPROACHES TO ALL CENTERS
AND INSTITUTES OF NIH.
THE GOAL OF REALIZING BENEFITS
OF BEHAVIORAL RESEARCH TOWARDS
HEALTH EQUITY.
THE CHARGE WAS VERY IMPORTANT
BEFORE AND BECAME MORE PROMINENT
AFTER THE PANDEMIC.
THE CHARGE WAS NOT TO TELL
SCIENTISTS WHAT TO DO BUT MODIFY
HOW WE DO IT.
AMONG THE RECOMMENDATIONS IS TO
HAVE MORE BEHAVIORAL AND SOCIAL
SCIENTISTS REPRESENTED IN THE
NIH WORKFORCE AT ALL LEVELS AND
INCLUDING IN STUDY SECTIONS AND
IN COUNCILS.
THE EXTERNAL PHENOMENON AND THE
CULTURE AND LIVED EXPERIENCES
AND SOCIAL REGULATIONS AND
PROGRAMS AND POLICIES SHOULD BE
CONSIDERED WHEN WE AIM TO STUDY
THE HEALTH OF ALL PEOPLE.
THE NICHD ALREADY HAD IT IN THE
MISSION AND VISION BUT
ENCOURAGING TO IMPLEMENT AT ALL
LEVELS WAS QUITE ENCOURAGING TO
SEE THAT THAT'S WHAT THE
CONGRESS WAS MANDATED TO THE
NIH.
AGAIN, THIS WAS A UNIQUE
OPPORTUNITY FOR ME TO SEE OUR
DIVERSE VIEWS WELL REPRESENTED.
SO IN CLOSING, I THINK MY
REFLECTION LIKE MANY OF US IS
GOING TO BE A WONDERFUL AND RICH
LEARNING EXPERIENCE.
I FELT HONORED TO BE IN THIS
COMPANY.
I WANT TO THANK DR. BIANCHI AND
THE STAFF WHO RUN THE COUNCIL.
THANK YOU FOR YOUR PATIENCE IN
TRANSITIONING ALL OF TO US
VIRTUAL AND IN PARTICULAR I WANT
TO THANK MY BUDDY, REBECCA CLARK
AND THE TEAM IN THE POPULATION
DYNAMICS BRANCH IN NICHD FOR
FRESHMAN COUNCIL MEMBERS THE
BODY IS THE GREATEST INVENTION
YOU ALL CAME UP WITH IN COUNCIL.
HAVING A BUDDY IS FANTASTIC.
OUR EXPERIENCE IN PERSON WITH
OUR TEAMS WAS FABULOUS WHEN WE
COULD DO IT IN PERSON.
TOO BAD WE COULDN'T DO IT
ANYMORE.
FOR THE REST OF YOU ALL IN
COUNCIL, THANK YOU SO MUCH FOR
OUR TIME TOGETHER IN THIS
IMPORTANT SERVICE.
THANK YOU.
>> THANK YOU FOR THE CRUCIAL
COMMENTS.
I'M GLAD IT WAS A POSITIVE
EXPERIENCE AND THANK YOU FOR
REPRESENTING THE COUNCIL OF
COUNCILS SUBGROUP.
TO BE CONTINUED.
WE HOPE BY JUNE WE'LL BE ABLE TO
HAVE IT IN PERSON WHICH WOULD BE
GREAT.
NEXT BUT NOT LEAST IS
DR. WYNSHAW-BORIS.
>> I'LL TRY NOT TO REPEAT MYSELF
TOO MUCH BUT IT'S HARD BECAUSE
WE ALL HAD SIMILAR GREAT
EXPERIENCES BECAUSE THE WORK OF
THE INSTITUTE IS SO IMPORTANT TO
ALL OF UP AND SEEING THE
IMPLEMENTATION OF THE PLAN.
IT WAS GREAT TO SEE THE NEW
PEOPLE BROUGHT IN AND GREAT TO
HEAR THE AREAS DISCUSSED AT THE
MEETINGS.
TO ME THIS IS ALWAYS THE BEST
PART OF THE MEETING AND WHAT A
DIVERSITY OF AREAS WE HAVE.
AND TO BE ABLE TO BE IN A
VIRTUAL ROOM WITH EXPERTS
THROUGHOUT THE WORLD HAS BEEN
TREMENDOUS.
I'VE ALSO APPRECIATED THE
INTERACTIONS WITH NOT JUST
COUNCIL MEMBERS BUT OF COURSE
THE GRANT PEOPLE AND LEADERSHIP.
I WISH MORE WOULD HAVE BEEN IN
PERSON AND IT INCLUDES FORMER
COUNCIL MEMBERS AS WELL AS
BECAUSE MANY HAVE NOT MET IN
PERSON AND SOME HAVE ONLY MET IN
COUNCIL MEETINGS AND A FEW OF US
DIANA AND CLIFF TAVEN THAT I
HAVE KNOWN MY ENTIRE SCIENTIFIC
CAREER AND MARTY, IT'S GREAT TO
SEE YOU GUYS AND WISH WE HAD
DONE MORE IN PERSON-MEETINGS
BECAUSE THAT'S SOMETHING I
MISSED.
SINCE TWO OF YOU COMMENTED YOU
HAD COVID MAYBE IT WAS A WISE
CHOICE NOT TO HAVE THIS IN
MEETING BUT I WISH WE COULD HAVE
BEEN THERE ANYWAY.
I THINK WE ALL HAVE OUR OWN
INDIVIDUAL AREAS OF EXPERTISE
AND I THINK IT WAS BROUGHT UP
FOR MOSTLY DEVELOPMENTAL BIOLOGY
AND GENETICS.
I WANT TO POINT OUT WHAT I THINK
ARE IMPORTANT THINGS CHILD
HEALTH DEVOTES ITSELF TOO AND
GREAT OPPORTUNITIES AND
CHALLENGES.
MATERNAL FETAL AND CHILDHOOD
HEALTH WERE OFTEN OVERLOOKED BY
OTHER INSTITUTES.
IT WASN'T THE CASE WITH LINDSEY
TODAY AND EACH OF THE TIMES WE
INTERACT THE INSTITUTE LEADERS I
THINK IT'S A GREAT THING AND THE
INSTITUTE OF CHILD HEALTH AND
DIANA, YOU HAVE TO MAKE SURE
YOU'RE FRONT AND CENTER WITH ALL
THESE TOPICS IS IMPORTANT.
WE HEARD ABOUT THINGS YOU'RE
DOING TO LEAD THE INSTITUTES BUT
AS THINGS GET LEFT OUT AND AS A
GENETICISTS THE BIG PROJECT IS
ALL OF US BUT IT DOESN'T INCLUDE
CHILDREN ONLY ADULTS.
AND CHILDREN'S HEALTH IS REALLY
IMPORTANT AND CHILDREN'S
SEQUENCING AS WELL.
WE WANT IT TO BE ALL OF US AND
THEY DON'T HAVE INFRASTRUCTURE
TO TAKE CHILDREN SO I'M HAPPY
YOU'RE LEADING A GROUP THAT'S
GOING TO FIGURE OUT WAYS TO MAKE
SURE THAT MOVES FORWARD.
IT'S IMPORTANT THE CHILD HEALTH
IS INVOLVED AND THE OTHER IS
DEVELOPMENT BIOLOGY AND MATERNAL
FETAL MEDICINE TO CHILDHOOD.
I THINK YOU PROBABLY ALL THINK
ABOUT THIS OCCASIONALLY BUT FOR
ME IT SEEMS MANY PROBLEMS AND
ISSUES WE SEE EVEN IN ADULTHOOD
HAVE THEIR ONSET IN CHILDHOOD
AND EVEN FETAL LIFE.
YOU HAVE TO LOOK BACK TO THESE
TIME POINTS FOR THINGS THAT
HAPPEN IN ADULT AND THINGS THAT
ARE IN IN ALS AND THINGS
INVOLVED ARE IN THE FETUS FOR
THINGS THAT AFFECT NEWBORNS,
CHILDREN OR AS WE HEARD FROM OUR
PATIENT TODAY AS SHE GETS MORE
AND MORE INTO ADULTHOOD.
THESE ARE THINGS ARE HAPPENING
EARLY ON SO THIS IS A VERY
IMPORTANT AREA.
WE'VE SEEN ROW PROPOSALS WITH
MODEL SYSTEMS AND HAVE TO
CONTINUE TO LOOK AT THE HUMANIST
MODEL SYSTEM AND I HOPE YOU GUYS
SEE MORE EMBRYONIC STEM CELL
WORK TO GET BACK TO THE HUMAN ON
SOME OF THESE ASPECTS AS WELL.
I HOPE WE GET BACK TO MORE IN
UTERO THERAPIES EITHER SOMEONE
WRITING GRANTS TO CHILD HEALTH
AND PROBABLY THE MOST IMPORTANT
CHALLENGE IS WE'RE IN THE
POST-DOBS DITION AND THE
RESEARCH AND IMPORTANCE TO ALL
OF US ARE BEING CHALLENGED AND
THAT'S MATERNAL FETAL MEDICINE
AND REPRODUCTIVE FREEDOM OF
CHOICE AND AS I SEE IT ALL THE
TIME IN TERMS OF WOMEN AND
FAMILIES FACED WITH DIFFICULTY
DECISIONS AND NOW THEY'RE
PREVENTED FROM DOING THESE
THINGS.
IT'S KIND OF -- I'M SORRY, I
ENDED WITH THAT ONE BUT IT'S A
CHALLENGE WE ALL HAVE TO MAKE
SURE WE PUSHBACK AGAINST AND
MAKE SURE WE HELP TO TRY TO
SOLVE.
I WANT TO THANK ALL OF YOU FOR
BEING ON COUNCIL WITH ME AND
THANK YOU, DIANA IN PARTICULAR
FOR YOUR LEADERSHIP AND WAIT TO
HEAR GREAT THINGS.
I WON'T FEEL BADLY ABOUT FILLING
OUT THE MYRIAD OF FORMS SEEMS
LIKE EVERY TWO TO THREE MONTHS
EVEN AFTER WE GET VETTED BUT
THAT'S ANOTHER POINT.
THANK YOU, AGAIN, DIANA.
>> THANK YOU, TONY.
WE HAVE PROTOCOLS WITH CHILDREN
BUT GENERALLY OVER 3 YEARS OLD
AND THAT'S A PROBLEM BECAUSE IF
WE WANT GENE THERAPY TO TREAT
INFANTS WITH DEGENERATIVE
DISEASE WE WAIT 3 YEARS IT'S TOO
LATE AND WE LOOK AT THE
INFRASTRUCTURE WE'LL NEED TO
MAKE THAT WORK.
THANK YOU FOR YOUR SERVICE AND
COMMENTS.
NEXT McN -- MS. NEUBERGER.
>> IT'S BEEN A PLEASURE TO WORK
WITH THIS GROUP OF COLLEAGUES.
I'VE SERVED ON THE COUNCIL THE
PAST FOUR YEARS AND ALWAYS
IMPRESSED BY THE PROFESSIONALISM
AND KNOWLEDGE OF THE NICHD STAFF
PROGRAM ADMINISTRATORS AND
DR. BIANCHI.
IT'S BEEN AN INCREDIBLE
EXPERIENCE.
SO REWARDING FOR ME.
I AM NOT A CLINICIAN OR
RESEARCHER.
I WAS A NURSE AND WENT TO LAW
SCHOOL.
I'VE BEEN PRACTICING LAW ABOUT
34 YEARS BUT I DO WORK FOR A
PEDIATRIC HEALTH SYSTEM AND WHAT
GETS DISCUSSED IS RELEVANT TO
WHAT WE DO AS A HEALTH SYSTEM
AND INTERESTING.
I APPRECIATE ALL THE KNOWLEDGE
AND UNDERSTANDING OF THE
CRITICAL RESEARCH BEING DONE.
I HOPE IN SOME WAY I'VE BEEN
ABLE TO CONTRIBUTE AND PROVIDE
GOOD FEEDBACK TO THIS COMMUNITY.
IN TERMS OF FEED BACK OVER ALL.
THE PATIENT'S STORIES GIVE US A
GLIMPSE TO THE CHALLENGES THESE
PEOPLE EXPERIENCE AND TO ME
THAT'S VERY SPECIAL.
THE INSTITUTE HAS DONE A GREAT
JOB ORIENTING US.
I CAN'T BELIEVE IT'S BEEN FOUR
YEARS.
I WOULD HAVE LOVED MORE
IN-PERSON MEETING DURING THE
PANDEMIC BUT IT WAS TOO
CHALLENGING AND THE ORIENTATION
YOU GAVE US WAS EXTRAORDINARY.
THE BUDDY SYSTEM HAS BEEN
FANTASTIC.
I WANT TO SEND A SPECIAL THANKS
TO DR. ROSLYN KING.
SHE'S BEEN SUCH A WONDERFUL
BUDDY TO ME.
THE CONSISTENTLY CHECKS IN
MAKING SURE I HAVE THE
INFORMATION AND ASSISTANCE ANDS
I NEED TO PARTICIPATE IN A
MEANINGFUL WAY AND THANK YOU ALL
FOR THIS INCREDIBLE OPPORTUNITY
AND THE VALUABLE WORK AND THE
WORK FOR OUR PEDIATRIC
POPULATION.
IT'S BEEN A GREAT EXPERIENCE.
>> YOU'VE BROUGHT A UNIQUE
PERSPECTIVE TO COUNCIL AND
APPRECIATE YOUR EFFORTS AND TIME
SPENT.
>> THANK YOU.
>> WE'LL GIVE THE LAST WORD TO
DR. ALAN TITA.
I SEE YOUR MEDICAL SCHOOL HAS A
NEW NAME.
I WASN'T AWARE OF THAT.
>> WE HAD A GREAT ACCOMPLISHMENT
AND IT'S BEEN FANTASTIC FOR THE
MEDICAL SCHOOL.
DIANA, THANK YOU VERY MUCH.
HE'S GRATEFUL FOR THE
OPPORTUNITY AND HONOR TO SERVE
ON COUNCIL AN HAS THOROUGHLY
ENJOYED IT AND FRANKLY DID NOT
NOTICE THE TIME GO BY.
I FEEL LIKE A CHILD THIS
INSTITUTE AND SERVED AS A P.I.
IN THE NETWORK CENTER AND ALSO
GOETH -- WORKING IN A GLOBAL
NETWORK AND DISORDERS NETWORK
AND GETTING A GOOD VIEW OF NICHD
WORKING ON PANELS AND REVIEW
WORKSHOPS AND THE CONFERENCE.
THIS HAS BEEN THE HIGHLIGHT FOR
ME.
I APPRECIATE THE DIFFERENT BALLS
NICHD HAS JUGGLED TO ACCOMPLISH
ITS MISSION.
SOMETIMES WE JUST LOOK AT IT
FROM OUR OWN PERSPECTIVE BUT
IT'S BEEN TERRIFIC TO SEE WHAT'S
GOING ON.
AND THERE'S BEEN A STRATEGIC
PLAN AND UNDERSTANDING AND
ADDRESSING THE PROBLEMS AND
AFFECTS OF THE PANDEMIC.
IN PREGNANT PEOPLE AND CHILDREN
AND IN ALL POPULATIONS.
I'M HEARTENED BY TRANSFORMATIVE
PROGRAMS COMING UP FROM NICHD
INCLUDING THE MATERNAL CENTERS
OF EXCELLENCE AND LOOKING AT
SOCIAL DETERMINATES OF HEALTH
AND COMMUNITY ENGAGEMENT.
THAT'S A NEW IMPORTANT FRONTIER
NICHD IS MOVING INTO.
AND LOOKING AT NETWORKS AND
GREAT TO SEE THE CONTINUAL
ENGAGEMENT IN GLOBAL HEALTH
WHICH HAS BEEN AMAZING AND
THERE'S BEEN CHANGES AND NEW
PEOPLE COMING TO NICHD.
I WANTED TO CONGRATULATE YOU FOR
A TERRIFIC JOB AND
CONGRATULATIONS THE ENTIRE NICHD
TEAM FOR THIS WORK DURING
CHALLENGING TIMES AND I HAVE
RESPECT FOR THE WORK AND THE
INDIVIDUALS.
THANK YOU TO MY COUNCIL BUDDY.
I IF I MAY GO OUT WITH ONE PLUG
IT'S TO CONTINUE TO ADVOCATE FOR
A SHORT REVIEW CYCLE FOR THE NEW
MECHANISM TO CONDUCT STUDIES IN
THE CLINICAL TRIALS NETWORK TO
MAKE THINGS NIMBLE.
RECENTLY AN PERSON HEALTH
EQUITIES RESEARCH NETWORK ON
MATERNAL AND INFANT DISPARITIES
PROGRAM TOOK MONTHS TO UNDER GO
TWO LEVELS TO AWARD NETWORKS.
MAYBE THE PROCESS MIGHT OFFER
SOME AVENUES TO CONTINUE THAT
CONVERSATION.
I WANT TO THANK ALL MEMBERS OF
COUNCIL.
IT'S BEEN A GREAT OPPORTUNITY TO
LEARN FROM YOU ALL AND THOSE WHO
CONTINUE TO CARRY THE BALL.
GOOD LUCK.
SEVERAL YEARS AGO I WAS SPEAKING
AT AN NICHD RETREAT.
MY MANTRA AT THE TIME WAS THE
P.O. WAS YOUR FRIEND AND I FEEL
THE TIME ON COUNCIL HAS BEEN
ATTENTIVE AND MY NEW MANTRA IS
NICHD IS OUR FRIEND AND LOOK
FORWARD TO HELPING IN ANY WAY AS
THE OPPORTUNITY ARISES.
THANK YOU VERY MUCH.
AND YOUR ADVOCACY HAS BEEN
IMPORTANT AS WE KNOW PEOPLE HAVE
EXPERIENCE THE COVID PANDEMIC
DIFFERENTLY AND MORE SEVERELY
PARTICULARLY IF THEY HADN'T BEEN
VACCINATED.
I KNOW ALL OF YOU HAD BEEN
INCREDIBLY ENGAGED AND IT'S BEEN
A JOY HAVING YOU ON OUR ADVISORY
COUNCIL.
WE KNOW IT DOES TAKE YOUR TIME
BUT I'M GLAD TO HEAR EVERYONE
GOT SOMETHING OUT OF IT.
AND HAVE AN EXPANDED
APPRECIATION FOR OUR PORTFOLIO
AND SOME OF THE THINGS WE
GRAPPLE WITH AND WE CAN TALK
MORE ABOUT THAT TOMORROW IN THE
CLOSED SESSION.
NOW I'LL TURN THE MEETING BACK
OVER TO REBEKAH.
WE'RE ALMOST IN THE HOME
STRETCH.
WE'RE GOING TO TALK ABOUT THE
CONCEPT CLEARANCE PORTION OF THE
MEETING.
AGAIN, THANK YOU EVERYONE AND
OVER TO REBEKAH.
>> ALL RIGHT.
THANK YOU SO MUCH.
WE HAVE SEVEN CONCEPTS FOR YOUR
CONSIDERATION.
I'LL ASK THE STAFF TO PROVIDE A
BRIEF OVERVIEW OF THE INITIATIVE
AND ASK COUNCIL FOR ANY
DISCUSSION.
AT THE CONCLUSION OF THE
DISCUSSION I'LL ASK COUNCIL
MEMBERS WHETHER THEY CONFIRM AND
WE'LL TAKE A VOTE AND THEN WE'LL
MOVE ON.
THE VERY FIRST CONCEPT IS FROM
THE MATERNAL PEDIATRIC
INFECTIOUS DISEASE BRANCH AND
IT'S ALL USING ARCHIVE DATA AND
SPECIMEN COLLECTIONS TO ADVANCE
MATERNAL AIDS RESEARCH.
>> IT'S SEEKING COUNCIL APPROVAL
FOR USING DATA IN SPECIMEN
COLLECTIONS TO ADVANCE MATERNAL
AND PEDIATRIC HIV RESEARCH AND
ADDRESS THE NEED FOR DATA
SHARING AND TRANSLATION OF
RESEARCH TO ANSWER IMPORTANT
QUESTIONS IN MATERNAL AND
PEDIATRIC HIV AND LOOK AT
STUDIES BY SUPPORTING SECONDARY
ANALYSIS OF THE COLLECTED DATA
AND SPECIMENS.
THE NICHD REPOSITORY HAS
ARCHIVED SAMPLES READY FOR
RESEARCH AND INCLUDING THE ATM,
FACTS AND IMPACT.
THE NICHD DATA AND SPECIMEN HUB
DASH PROVIDES ACCESS TO THE LINK
TO THE SAMPLES.
WE SEEK INVESTMENT IN THE
SPECIMEN REPOSITORIES WITH THE
FUNDING OPPORTUNITY ANNOUNCEMENT
AND RESOURCES ARE ENCOURAGED TO
USE ARCHIVED SPECIMENS TO
GENERATE NEW FINDINGS AND
ANSWERS TO QUESTIONS OF THE
OFFICE OF AIDS RESEARCH.
WE'D LIKE TO CONTINUE THE
INITIATIVE AS ALREADY FUNDED
OVER 1,000 PROJECT THAT ALLOW
FOR THE EXPANSION OF THESE
AFFECTS.
>> ANY COMMENTS OR QUESTIONS?
HEARING NO COMMENTS, SEEING NO
HANDS, DO I HAVE A MOTION TO
APPROVE THE CONCEPT?
DR. MALDONADO DID YOU HAVE A
COMMENT?
>> YEAH.
I WANTED TO SUPPORT THIS
CONCEPT.
IT'S CRITICAL ESPECIALLY TO PULL
TOGETHER THE MANY YEARS AND
DOLLARS SPENT AND TIME SPENT ON
THESE DATA SETS AND REPOSITORIES
TO REALLY BRING TOGETHER A NEW
GENERATION OF RESEARCH.
AND INTEGRATING IN THE NEXT
GENERATION OF RESEARCH CAPACITY
IS A USEFUL WAY TO LEVERAGE
EXISTING RESOURCES WITH A
MODICUM OF EXPENSE.
>> DO I A NOTION -- MOTION TO
APPROVE.
>> SO MOVED.
>> SECOND.
>> USE YOUR ACTION BUTTONS TO
VOTE YES OR NO.
>> THE NEXT CONCEPT WE'LL HEAR
FROM DR. ALAN.
A NEW MEMBER OF THE
CONTRACEPTION RESEARCH BRANCH
DEVELOPMENT OF NOVEL
CONTRACEPTIVE METHODS.
>> THE PROGRAM SEEKS COUNCIL
APPROVAL FOR AN INITIATIVE
ENTITLED DEVELOPMENT OF NON
STROIDAL CONTRACEPTIVE METHODS
AND THERE'S TREMENDOUS NEED FOR
THIS FOR MEN AND WOMEN AND THIS
CONTINUES THE PARADIGM SHIFT
TOWARDS MORE SPECIFIC
CONTRACEPTIVE MODALITIES WITHOUT
A STEROIDAL MECHANISM AND IT
ALLOWS FOR FEMALE OR MALE METHOD
DEVELOPMENT AS WELL AS
DEVELOPMENT OF DELIVERY DEVICES
IN MULTI-PURPOSE PREVENTION
TECHNOLOGIES WITH CONTRACEPTIVE
AND ANTI-INFECTIVE CAPABILITY.
THE NICHD REMAINS THE LEADING
FUNDER OF EARLY STAGE
CONTRACEPTIVE RESEARCH FOR
MULTI-PURPOSE TECHNOLOGIES
THROUGHOUT THE WORLD AND
CONTINUES FUNDING IN THIS AREA
IF IS OF PARAMOUNT IMPORTANCE OF
THESE FIELD.
IN THE PREVIOUS ITERATION OF THE
INITIATIVE THE 61R33 HIGHLY
INNOVATIVE CONTRACEPTION
PROGRAMS WERE DEVELOP FOR MALE
AND FEMALE AND PREVENTION
TECHNOLOGY.
FIVE OF THE EIGHT PROGRAMS
TRANSITIONED TO THE R33 PHASE
AND TWO MALE AND THREE FEMALE.
THE CONCEPT ALLIANCE WITH THE
STRATEGIC PLAN IS IN THE
REPRODUCTIVE HEALTH AND
DEVELOPMENT OF SAFE AND
EFFECTIVE THERAPEUTICS AND THE
CONTRACEPTION BRANCH PRIORITY.
THANK YOU.
>> QUESTIONS OR COMMENTS?
HEARING NONE DO I HAVE A MOTION
TO APPROVE THE CONCEPT?
>> I MAKE A MOTION TO APPROVE
THE CONCEPT?
>> SECOND.
>> SECONDED.
>> ALL IN FAVOR PLEASE USE YOUR
VOTING BUTTON.
>> WE HAVE 14 YESES.
>> THANK YOU SO MUCH.
THE NEXT PERSON IS DR. CANDACE
TAILIN AND SHE'LL BE PRESENTING
CENTERS FOR RESEARCH ON HEALTH
DISPARITIES IN UTERINE FIBROIDS.
>> THE INITIATIVE WOULD LOOK AT
BASIC, CLINICAL AND
TRANSLATIONAL RESEARCH.
THE ULTIMATE GOAL OF THE PROGRAM
IS TO IMPROVE MODIFIABLE ASPECTS
OF THIS COMMON GYNECOLOGIC
CONDITIONS AND THERE'S SYMPTOMS
OF BLEEDING, INFERTILITY AND
ADVERSE PREGNANCY OUTCOMES AS
WELL AS SIGNIFICANT IMPACT ON
QUALITY OF LIFE.
FIBROIDS ALSO REPRESENT THE
LARGEST HEALTH DISPARITY IN G
GINE -- GYNOCOLOGIC HEALTH AND
BLACK WOMEN ARE SEVEN TIMES MORE
LIKELY TO HAVE AN MYOMECTOMY.
AND THERE'S A PATH TO GREATER
EQUITY AND FOR TREATMENT AND
CURE.
THE INITIATIVE IS RESPONSIVE TO
PRIORITIES ACROSS THREE
BRANCHES.
THE IMPORTANT UNITE AND STRIVE
INITIATIVE AND THE NICHD
STRATEGIC PLAN.
THANK YOU.
>> ANY COMMENCES OR QUESTIONS.
IS THERE A MOTION TO APPROVE?
>> MOTION TO APPROVE?
>> SECOND.
>> ALL IN FAVOR?
>> WE HAVE 14 YESES.
>> WE KNOW MOVE ON TO THE
DEVELOPMENT BIOLOGY AND
STRUCTURAL VARIATION BRANCH
PRESENTING OPPORTUNITIES FOR
ADVANCING REGENERATION RESEARCH.
>> THE PROGRAM SEEKS COUNCIL
APPROVAL FOR A NEW INITIATIVE
FOR OPPORTUNITIES FOR
REGENERATION RESEARCH.
THIS INITIATIVE ADDRESSES THE
ASPIRATION GOAL OF ADVANCING THE
ABILITY OF REGENERATIVE GAINS TO
GET THE BODY'S OWN PROCESSES.
THE DEVELOPMENT AND VARIATION
BRANCH AND THE NATIONAL CENTER
FOR REHAB RESEARCH PUBLISHED A
REQUEST FOR INFORMATION AND
ORGANIZED COMMUNITY ON THIS
TOPIC.
EACH YEAR ABOUT 1 IN 2,000
BABIES ARE BORN WITH DEFECTED
AND MORE THAN 1. 6 MILLION FACE
AMPUTATION.
WHILE SOME HAVE THE ABILITY TO
REGROW AMPUTATED LIMBS IN SOME
MAMMALS THE VARYING BRANCH
PROPOSES TO DO BASIC RESEARCH IN
ANIMAL MODELS TO ADDRESS THE
CRITICAL GAPS IN OUR
UNDERSTANDING OF KEY ASPECTS OF
THE REGENERATION PROCESS AND
TISSUE REGENERATION TO GAIN
INSIGHT ON PROCESSES THAT CAN BE
MANIPULATED TO PROMOTE LIMB
REGENERATION INCLUDING IN
MAMMALS THAT MIGHT OTHERWISE
LACK THE ABILITY FOR
REGENERATION.
THANK YOU.
>> ANY QUESTIONS OR COMMENTS?
>> I THINK IT'S A TIMELY CONCEPT
TO BRING UP BECAUSE THERE'S A
LOT KNOWN ABOUT LIMB
REGENERATION AND THE TIME IS
RIGHT FOR UNDERSTANDING THESE
TECHNOLOGIES AND REGENERATION IN
MODEL ORGANISMS FOR THIS TO BE
ADVANCED.
IT WOULD BE AN EXAMPLE OF USING
REGENERATIVE MEDICINE TECHNOLOGY
FOR THE FUTURE.
I'M SUPPORTIVE OF THIS.
>> ANY OTHER THOUGHTS?
THERE'S BEEN A MOTION TO APP
APPROVE.
>> SO MOVED.
>> SECOND.
>> PLEASE GO AHEAD AND VOTE.
>> WE HAVE 15 YESES.
>> NEXT IS THE CRITICAL ILLNESS
BRANCH PRESENTING PEDIATRIC
CRITICAL CARE AND SCIENTIST
DEVELOPMENT PROGRAM.
>> THANK YOU.
PROGRAM SEEKS COUNCIL APPROVAL
FOR AN INITIATIVE ENTITLED THE
SCIENCE DEVELOPMENT PROGRAM.
THE PURPOSE OF THIS NATIONAL
PROGRAM IS TO PROVIDE CAREER
DEVELOPMENT SUPPORT FOR JUNIOR
PEDIATRIC CRITICAL CARE
PHYSICIANS OR TRAUMA SURGEONS.
GIVEN THE DEMANDS OF TRAINING
AND THE ESTABLISHED RESEARCH
MENTORS IN THE FIELD IT'S
CHALLENGING TO MAINTAIN A
WORKFORCE OF WELL REPAIRED
PHYSICIAN SCIENTISTS.
THE PROGRAM WAS ESTABLISHED IN
2004.
THE PROGRAM WAS HIGHLY
SUCCESSFUL.
SINCE THE INCEPTION THERE'S BEEN
55 SCHOLARS AND 62% WENT ON TO
INDEPENDENT K FUNDING AND 35%
RECEIVED ALL U-LEVEL FUNDING.
DESPITE THE SUCCESS OF THE
PROGRAM THE DATA REMAINS SMALL.
THE GOAL OF THIS INITIATIVE IS
TO TRAIN JUNIOR INVESTIGATORS TO
PICK UP RESEARCH TO IMPROVE
OUTCOMES AMONG CRITICALLY ILL
AND INJURED PROGRAM AND THE
CONCEPT ALIGNS WITH THE NICHD'S
STRATEGIC PLAN AND PRIORITIES OF
THE TRAUMA AND CRITICAL ILLNESS
BRANCH TO ADVANCE THE CARE AND
TREATMENT OF OUR MOST VULNERABLE
CHILDREN.
THANK YOU.
>> CLEARLY THREERE'S A NEED FOR
STRONGER RESEARCH BASE AND THE
STATISTICS YOU CITED IS A STRONG
ENDORSEMENT AND I'M SUPPORTIVE.
AND THERE'S THERE'S A POINT
ABOUT THE PROTECTED TIME.
>> 35%.
>> IF THERE'S NO OTHER QUESTIONS
OR COMMENTS IS THERE A MOTION TO
APPROVE?
>> MOTION TO APPROVE.
>> SECOND.
>> PLEASE VOTE.
>> WE HAVE 15 YESES.
>> THANK YOU SO MUCH.
>> NEXT IS THE OBSTETRIC AND
PEDIATRIC BRANCH.
>>
GOOD AFTERNOON.
WE SEEK AN INITIATIVE ENTITLED
TRANSLATIVE RESEARCH AND
PHARMACOLOGY AND THERAPEUTICS.
THERAPEUTICS DISCOVERY AND
DEVELOPMENT FOR PEDIATRIC AND
PREGNANT AND LACTATING PATIENTS
LAGS BEHIND FOR NON-PREGNANT
POPULATION.
RESEARCH ON NEW AND RE-PURPOSED
THERAPEUTICS AND MODELS AND
OTHER TECHNOLOGIES THAT EXPLAIN
THE UNDERLYING MECHANISMS OF
DRUG ACTIONS ARE NECESSARY TO
DRIVE THE TREATMENT OF THESE
POPULATIONS.
THE LAST CYCLE OF THE INITIATIVE
WE FIND A 17 AWARDS FOR RESEARCH
ON NEW AND REPURPOSED
THERAPEUTICS MODELS AND
BIOMARKERS AMONG OTHERS.
THE PURPOSE OF THIS INITIATIVE
IS TO PROMOTE BASIC
TRANSLATIONAL AND CLINICAL
RESEARCH TO FIRST ADVANCE THE
UNDERSTANDING OF THE UNDERLYING
MECHANISMS OF DRUG ACCIDENTS AND
SECOND, TO DISCOVER NOVEL
THERAPEUTICS AND ENHANCE THE USE
OF THE EXISTING DRUGS OR DRUG
REPURPOSING FOR SAFER AND MORE
EFFECTIVE MEDICATIONS IN
CHILDREN AND PREGNANT AND
LACTATING PERSONS.
THIS ALIGNS WITH THE NICHD'S
STRATEGIC PLAN AND ALIGNS WITH
NUMEROUS RECOMMENDATIONS MADE BY
THE TASK FORCE.
THANK YOU.
>> THANK YOU.
OTHER QUESTION OR COMMENTS FROM
THIS CONCEPT?
NOT HEARING ANY, IS THERE A
MOTION TO APPROVE.
>> MOTION TO APPROVE.
>> A SECOND?
>> SECOND.
>> PLEASE GO AHEAD AND VOTE.
>> WE HAVE 15 YESES.
>> THANK YOU.
THE LAST CONCEPT IS FROM
DR. PARIZI CHIEF OF THE
INTELLECTUAL DISABILITIES BRANCH
ON INCLUDE PROJECT INITIATIVES.
>> THANK YOU.
PROGRAM SEEKS COUNCIL APPROVAL
FOR INITIATIVES UNDER THE
INCLUDE PROJECT INVESTIGATION OF
CO-CURRENT CONDITIONS TO
UNDERSTAND DOWN SYNDROME.
IT'S THE MOST COMMON CAUSE OF
INTELLECTUAL DISABILITIES AND
INDIVIDUALS FACE SIGNIFICANT
CHALLENGE.
IT WAS LAUNCHED TO UNDERSTAND
THE DOWN SYNDROME ON BASED ON
STUDIES AND ASSEMBLY OF A LARGE
COHORT OF INDIVIDUALS WITH DOWN
SYNDROME ACROSS THE LIFE SPAN
AND THREE, INCLUSIVE CLINICAL
TRIALS OF EXISTING AND FUTURE
TREATMENTS.
SINCE 2019 NIH HAS INVESTED MORE
THAN $183 MILLION IN 200
PROJECTS AND SUPPORTED NUMEROUS
TRAINING AWARDS.
A DATA KOOSHED -- THE
THE CONCEPT WILL ALLOW
INITIATIVES TO MEET THE GOALS OF
THE INCLUDE PROJECT.
IN FISCAL YEAR 23 THERE WAS A
LARGE COHORT STUDY COLLECTING
DATA FROM DIVERSE COMMUNITIES
AND A BIO BANKING INITIATIVE TO
ENSURE SPECIMENS ARE COLLECTED
IN A UNIFORM MANNER FOR BROAD
DISTRIBUTION.
INCLUDE IS BEING DEVELOPED BY
THE OFFICE OF THE OF NIH AND
NICHD IS SERVING AS THE
INSTITUTE OF RECORD FOR
CLEARANCE FOR CONCEPTS THAT
ARISE WITHIN THE PROJECT.
THANK YOU.
>> ANY QUESTIONS ABOUT THE
INITIATIVE?
>> THINK THIS IS AN APPEAR
INITIATIVE AND A LARGE
INVESTMENT WITH A LARGE BENEFIT
TO THOSE WITH DOWN SYNDROME AND
INTELLECTUAL DISORDERS AND COULD
SERVE AS A MODEL.
THANK YOU.
>> I SECOND THAT VIEW.
>> DO WE HAVE A MOTION TO
APPROVE.
>> SECOND?
>> PLEASE GO AHEAD AND VOTE.
>> THANK YOU, COUNCIL MEMBERS,
REBEKAH.
WE'RE NOT TOO BAD OFF.
THIS CONCLUDES THE FIRST DAY OF
OUR TWO-DAY VIRTUAL NICHD
ADVISORY COUNCIL MEETING.
I WANT TO THANK YOU ALL FOR
ATTENDING.
I ALSO WANT TO GIVE SPECIAL
THANKS TO OUR TWO MEMBERS WHO
CONFESSED THEY'RE CURRENTLY
EXPERIENCING COVID SO WE
APPRECIATE YOUR WILLINGNESS TO
BE WITH US WHILE HOPEFULLY YOU
ARE RECOVERING.
I LOOK FORWARD TO SEEING ALL THE
COUNCIL MEMBERS TOMORROW FOR THE
CLOSED SESSION PART OF THE
MEETING.
THE START TIME WILL BE THE SAME
AS IT WAS TODAY, 12:00 P.M.
EASTERN DAYLIGHT TIME FOR
COUNCIL MEMBERS.
EXTRAMURAL STAFF SHOULD PLAN TO
JOIN US AT AROUND 1:15 P.M.
BECAUSE WE WILL START WITH A
CLOSED PRESENTATION ON THE
INTRAMURAL PROGRAM AND HOPE
EVERYONE HAS A GREAT EVENING AND
THANK YOU FOR STAYING WITH US
UNTIL THE END AND PARTICULARLY
THE CLOSING COMMENTS OF THE
GRADUATING COUNCIL MEMBERS.
HAVE A GREAT EVENING AND WE'LL
SEE YOU TOMORROW.