>>ALL RIGHT, WELL, EVERYONE IS
JOINING US ON ZOOM FOR THIS
COUNCIL MEETING--THROUGHOUT THE
NEXT 2 DAYS AND COULD I HAVE THE
FIRST SLIDE, KELLIE?
WANT TO JUST START OFF BY JUST
THANKING THE MEMBERS WHO ARE
ROTATING OFF.
AND THAT'S TOM CARMICHAEL, ANITA
[INDISCERNIBLE], AARON
[INDISCERNIBLE], ARNOLD
[INDISCERNIBLE], AND EILEEN
MURRAY, IT'S BEEN A REAL
PLEASURE WORKING WITH YOU AND WE
WORKED WITH EACH OF YOU PRETTY
HARD THROUGHOUT THE LAST COUPLE
OF YEARS.
SORRY, WE WEREN'T ABLE TO GET
TOGETHER AS MUCH AS WE WOULD
HAVE OTHERWISE IN PERSON, BUT
I'M SURE THAT WE'RE GOING TO BE
MIXING IT UP IN THE YEARS TO
COME.
SO THANK YOU SO MUCH FOR YOUR
SERVICE AND EXTEND THIS EXTRA
MEETING WAITING FOR THE NEW
MEMBERS TO BE OFFICIALLY
APPROVED.
WE HAVE A GREAT MEETING LINED UP
FOR YOU IN THE NEXT COUPLE OF
HOURS AND TOMORROW, AND NOW I
WOULD JUST LIKE TO HAND IT OVER
TO BOB.
>> OKAY, I WILL LET YOU ALL
APPRECIATE THAT SLIDE FOR A
SECOND.
WHY DON'T WE GO BACK TO
SEEING--GO BACK TO SEEING
EVERYBODY.
OKAY, SO I WOULD LIKE TO
ALSO--I'M BOB FINKELSTEIN, I
WOULD ALSO LIKE TO WELCOME YOU
TO THE OPEN SESSION, IT'S GREAT
TO SEE EVERYBODY, EVEN IF YOU'RE
IN YOUR RECK TANGLES.
JUST A REMINDER THIS IS BEING
VIDEOCAST LIVE AND IT'S ARCHIVED
SO JUST KEEP THAT IN MIND, TRY
TO LOOK AND BE YOUR BEST.
SO I THINK WE HAVE THESE VIRTUAL
MEDINGS DOWN BY NOW SO I WON'T
TALK ABOUT FORMAT.
I WILL JUST SAY THAT TIM LIDEN
HAS NOW PRETTY MUCH REPLACED
KELLIE AS OUR FULL-TIME POINT OF
CONTACT FOR COUNCIL WHICH IS A
HUGE, HUGE JOB OF BEFORE, DURING
AND AFTER COUNCIL AND TIM HAS
DONE A PECK TACKULAR JOB GETTING
UP TO SPEET.
IT'S AMAZING.
ALSO WE HAVE BROOK, SHE WILL BE
TAKING YOU IN AND OUT OF WAITING
ROOMS IF YOU'RE IN CONFLICT AND
HELPING IN MANY OTHER WAYS AND
BROOK IS ALSO TERRIFIC AND
KELLIE IS ALWAYS LURKING IN THE
BACKGROUND MAKING SURE THINGS
ARE OKAY, SO HELLO TO KELLIE.
JUST THE ONLY THING I WILL SAY
AS A REMINDER IS THAT IF YOU
WANT TO SAY SOMETHING OR MAKE A
MOTION, YOU CAN EITHER JUST
SPEAK UP, THAT'S FINE WITH ME,
OR YOU CAN RAISE YOUR EMOJI,
HAND, IT'S PROBABLY EASIER SO I
CAN KEEP TRACK OF WHO RAISED
THEIR HANDS, WHEN AND WHEN WE
HAVE MOTIONS, TIM WILL ENTER THE
MOTION INTO THE CHAT AND YOU
WILL BE VOTING IN THE CHAT.
YES, NO, OBLIGATIONS STAIN.
OKAY, INTRODUCTION NOW, FIRST
THING WE NEED IS THE FORMAL
APPROVAL OF MINUTES FROM THE
LAST MEETING WHICH IS IN THE
ECB, FIRST POST BEING SECTION OF
THE NANDS GBL INFO TAB, UNLESS
THERE'S A DESIRE TO DISCUSS
THESE MINUTES IN INCREDIBLE
DETAME, I WILL PAUSE FOR A
SECOND, I NEED A MOTION TO
APPROVE THE MINUTES AND A
SECOND.
>> SO MOVED.
>> THANK YOU, SECOND FROM LUIS.
>> AND TIM ENTERED THAT INTO THE
CHAT OR HE WILL BE.
>> SO YOU'LL BE VOTING ON THE
MOTION IN THE CHAT SO WHY AM I
NOT SEEING THAT?
>> OKAY, I DON'T WANT TO HOLD US
UP ON THE MINUTES SO ALL WHO
APPROVE OF THE MINUTE SAYS RAISE
YOUR HANDS.
ANY OPPOSED?
OKAY I'M GOING TO ASSUME THE
MINUTES ARE OPPOSED--APPROVED
AND MEAN WHILE WE'LL WORK OUT
WHAT'S GOING ON IN THE CHAT.
MAYBE I'M DOING SOMETHING WRONG
HERE.
THERE IT IS, OKAY.
OKAY, SO THE MINUTES ARE
APPROVED, THAT'S EXCITING.
SO IN TERMS OF ALSO UNDER THE
GENERAL INFORMATION CATEGORY IN
THE ECP, ALL THE COUNCIL
MEETINGS DATES THROUGH 2024 ARE
LISTED.
NOIA THEY WILL CONTINUE TO BE ON
WEDNESDAY AND THURSDAYS.
RIGHT NOW THEY'RE 1 TO
APPROXIMATELY 5:30 ON EACH OF
THOSE DAYS SO PLEASE HOLD THOSE
DATES, LET US KNOW IF YOU HAVE A
CONFLICT IN ADVANCE, WE'RE STILL
BY THE WAY TRYING VERY HARD TO
FIGURE OUT WHEN WE START HAVING
PERSON TO PERSON MEETINGS, NOT
TO MENTION, GOING OUT TO DINNER
TOGETHER, WHICH IS A LOT OF FUN
AND WE WILL GET BACK TO YOU AS
SOON AS WE KNOW MORE ABOUT THAT.
THAT COULD END UP BEING FOR NOW
ONCE A YEAR OR WHATEVER.
OKAY, I WILL MOVE ON TO THE EXIT
REVIEW PROCESS, EVERY ROUND, I'M
SUPPOSED TO REPORT TO YOU ON
THIS, THIS IS WHAT 3 DESIGNATED
COUNCIL MEMBERS, THIS TIME, I
THINK IT WAS KIM AND I THINK
JOHN WAS THE THIRD 1, AND
APPROVE A SUBSET OF APPLICATIONS
BEFORE COUNCIL MEETS.
THIS ALLOWS OUR GRANTS
MANAGEMENT BRANCH TO ISSUE THESE
AWARDS SOMETIME BEFORE COUNCIL
SO THE PROJECTS CAN GET STARTED
AS SOON AS POSSIBLE.
WE TYPICALLY FOCUS ON GRANTS IN
THE PAY LINE THAT HAVE NO
ISSUES.
AT THE MOMENT WE'RE ACTUALLY
FOCUSING ON R21S OR RO3S,
THERE'S ALSO RO-1s AS WELL, SO
THANKS TO KEN AND JOHN AND THERE
WERE 141 APPLICATIONS ELIGIBLE
TO EXPEDITED, FOR BUDGETEDARY
REASONS WE'RE NOW PAYING AND
POLICY REASONS, WE'RE NOW PAYING
FEWER OF THESE APPLICATIONS TO A
SEPTEMBER COUNCIL BUT MANY CASE,
WE ALREADY PAID A NUMBER OF THEM
AND A NUMBER OF THEM AND THE
OTHERS WILL BE ISSUED AS SOON AS
WE POSSIBLY CAN, AND AS I SAY,
EVERY TIME BUT IT BEARS
REPEATING THIS, OUR ABILITY TO
DO THIS SO QUICKLY IS A
TESTAMENT TO OUR REALLY AMAZING
GRANTS MANAGEMENT BRANCH, LED BY
SHELLY WILBOURN, NOT TO MENTION
OUR TERRIFIC PROGRAM STAFF WHO
HAVE TO GET EVERYTHING APPROVED
IN THEIR SYSTEM IN ORDER FOR
APPLICATIONS TO BE EXPEDITED.
SO IN THE OLD DAYS WHEN PEOPLE
USED TO GET TOGETHER IN ROOMS
AND HAVE MEETINGS AND WE HAD
MORE TIME, WE USED TO INTRODUCE
ALL OUR NEW STAFF, AT A COUNCIL
MEETING, WE HAVE LESS TIME,
WE'RE TRYING TO STREAMLINE THESE
VIRTUAL MEETINGS, SO IF SOMEONE
COULD JUST PUT UP THE SLIDE
LISTING THE NEW PEOPLE WHO ARE
JOINING NINDS, YOU CAN TAKE A
LOOK AT THAT AND YOU CAN ALSO
READ THEIR BIOS IN THE ECB,
THERE'S A LOT OF REALLY
INTERESTING PEOPLE THAT GET
HIRED HERE.
THERE IT GOES, SO CAN YOU SEE,
THERE'S NEW PEOPLE IN WALTER'S
OFFICE, CLINICAL RESEARCH,
DIVISION OF NEURAL RESEARCH,
NEUROSCIENCE AND MY OFFICE, THE
OFFICE OF EXTRAMURAL ACTIVITIES
AND DIVISION, AND ALSO IN THE
ONCE OFFICE, I WON'T GO THROUGH
ALL OF THEM BUT I ENCOURAGE YOU
TO READ ABOUT THEM IN THE ECB,
SO CAN YOU TAKE THAT SLIDE DOWN
I THINK THAT'S ALL I NEED TO SAY
SO I WILL TURN IT OVER TO WALTER
FOR HIS DIRECTOR'S TALK.
>>THANKS, BOB AND TO HELP SAVE
TIME AND ACTUALLY PRACTICE AND
GET THE TALK RIGHT AND I
RECORDED IT AGAIN AND SO WHY
DON'T WE SHOW THE DIRECTOR'S TALK
>>HELLO I'M DR. WALTER
KOROSHETZ, AND IT'S MY PLEASURE
TO PRESENT TO YOU THE REPORT, AT
THE 217th NINDS COUNCIL
CHANGES.
WANT TO TALK ABOUT CHANGES WITH
THE NINA SCHOR, AS BEEN
APPOINTED AS THE NEW NIH ACTING
DEPUTY DIRECTOR FOR INTRAMURAL
RESEARCH, SHE WILL REPLACE
DR. MICHAEL GOTTESMAN, WHO HAS
SERVED FOR THE PAST 29 YEARS.
AS YOU KNOW, DR. SCHOR WAS
PREVIOUSLY WORKING AS THE ACTING
SCIENTIFIC DIRECTOR SO WITH HER
DEPARTURE, WE APPOINTED DR. JEFF
DIAMOND AS THE ACTING SCIENTIFIC
DIRECTOR FOR THE THE INTRAMURAL
RESEARCH PROGRAM, HE'S BEEN AN
INVESTIGATOR AT NINDS FOR 23
YEARS, STUDYING HOW SYNAPSES AND
NEURONS AND SMALL CIRCUITS
PERFORM IN THE COMPUTATIONAL
TASKS REQUIRED FOR MAMMALIAN
RETINA AND WE ALSO APPOINTED AMY
BANY ADAMS, THE CURRENT NINDS
DEPUTY DIRECTOR FOR SCIENTIFIC
MANAGEMENT AND OPERATIONS TO BE
THE NINDS ACTING PRINCIPAL
DEPUTY DIRECTOR AFTER
DR. SCHOR'S DEPARTURE.
IN ADDITION THERE ARE SOME
LEADERSHIP CHANGES ACROSS NIH AT
THE OTHER INSTITUTES, DR. MONICA
BERTAGNOLLI, SURGEON AT THE
DANNA FARBER CANCER INSTITUTE
HAS BEEN SELECTED AS THE NEW
DIRECTOR OF THE NATIONAL CANCER
INSTITUTE, AND AS YOU PROBABLY
HEARD IN DECEMBER, DR. ANTHONY
FAUCI, ANNOUNCED HE IS STEPPING
DOWN FROM THE POSITION OF
DIRECTOR OF THE NIAID AND AS
WELL AS THE POSITION OF THE
CHIEF MEDICAL ADVISOR TO THE
PRESIDENT.
IN HIS 54 YEARS AT NIH,
DR. FAUCI HAS SERVED AS DIRECTOR
OF NIAID FOR 38 YEARS AND
ADVISED 7 U.S. PRESIDENTS WHO
HAD A NUMBER OF SERIOUS
INFECTIOUS DISEASE PANDEMICS AND
OTHER CONDITIONS.
AS I ALWAYS DO, START OFF BY
TALKING ABOUT THE BUDGET.
AS YOU CAN SEE HERE IN THE
SLIDE, NINDS HAS BEEN FORTUNATE
TO RECEIVE INCREASES IN ITS BASE
BUDGET OVER THE YEARS, SEEN HERE
IN 2018, LOOKING TO 2023,
HOWEVER, THERE'S 2 BUDGETS THAT
WE HAVE TO PAY ATTENTION TO, 1
IS THE HOUSE BUDGET, THE OTHER
THE SENATE BUDGET AND IN
ADDITION, THE EXPECTATION IS
THAT IT WILL NOT BE A BUDGET
UNTIL THE ELECTION AND
POTENTIALLY NOT EVEN TO LATE
WINTER, AND THAT WILL BE
OPERATING UNDER WHAT'S CALLED A
CONTINUING RESOLUTION, WHICH IS
THE SAME AMOUNT OF MONEY THAT
WE'VE GOT IN 2022, IS WHAT WE
WOULD PLAN FOR TO USE IN 2023.
SO WE'RE PLANNING FOR OPERATING
UNDER WHAT WE CALL THE C. R., WE
HAVE 2 OTHER BUDGETS TO LOOK AT.
IN THE 2023 HOUSE MARK, COME TO
US, THEREY'S A 10% INCREASE IN
THE NINDS BASE, ONLY 3.1% IN THE
SENATE MARK.
NOW THE NINDS, WE PRESENTED DOES
NOT INCLUDE THE FUND THAGOREAN
COMES TO NIH TO THE BRAIN
INITIATIVE OR THE HEAL
INITIATIVE TO HELP END ADDICTION
LONG-TERM.
WE NEED MUCH OF THE PAIN
RESEARCH, IF THOSE ADDITIONAL
APPROPRIATIONS ARE AT AN END AND
YOU SLEEP APNEA AND OBESITYY
THAT IN THIS RED BOX, TOTAL
NINDS APPROPRIATION, INCLUDING
THESE ADDITIONAL MONIES.
THAT WOULD GIVE 8.5% INCREASE
UNDER 2023, HOUSE MARKS AND
SENATE MARKS, I WANT TO MENTION
THAT THE HOUSE MARKS IS A
200 MILLION INCREASE TOTALS
JEIMER INITIATIVE AND AS YOU
KNOW NINDS, MANAGES ABOUT 10% OF
THOSE FUNDS AND THIS WOULD
INCREASE OWSH ADMIN RECORDS AND
MINISTRATION, THIS INITIATIVE
$294 MILLION A YEAR SO THAT'S
WHAT THE BUDGET LOOKS LIKE RIGHT
NOW.
AND WE BREAK THIS DOWN INTO
NONCOMPETING FUNDS, NONCOMPETING
MEANS THAT THIS MONEY HAS BEEN
OBLIGATED, THROUGH GRANTS THAT
MAY LAST 2, 3, 5 YEARS, WITH THE
OUT YEAR COURSE FOR GRANTS,
THAT'S GOING UP BY ABOUT
50 MILLION IN 2023, UNDER A
CONTINUING RESOLUTION, SO ALL
THESE NUMBERS ARE IN THE WORST
CASE SCENARIO, CONTINUING
RESOLUTION.
FOR COMPETING FUNDS, THE FUNDS
THAT ARE USED FOR NEW GRANTS
WITHIN THE PAY LINES AND THESE
ARE GRANTS AND MOSTLY RO-1s
AND R21S THAT COME IN UNDER THE
PARENT AWARD AND THEY GET TO
2023, WITH THE C. R., WE HAVE
LESS COMPETING FUNDS BUT WE WERE
ABLE TO FUND A NUMBER OF
PROJECTS NAMELY R21s, IN 2022
MONEY AND FULLY FUND THEM FOR
THEIR 2023 YEARS AND THAT
ACTUALLY IS WHAT ALLOWS US TO
KEEP THE 14th PERCENTILE IN
THE CASE OF A C. R., SCENEOT
NEXT SLIDE OF THE NONPAY LINE
INVESTMENTS AND THESE INCLUDE
EARLY STAGE INVESTIGATORS ABOVE
THE PAY LINE GRANTS, BRIDGES TO
MULTIPLE PIs, PROGRAM PRIORITY
GRANTINGS, THE R35 PROGRAM,
WHICH FUNDS INVESTIGATORS FOR 8
YEARS WITH A CAP OF 750 K AND
REMOVES THEM FROM THE RO-1 POOL
AND TRIALS, SUPPLEMENTATION
PROGRAM AND ADDITIONAL FOARE
As AND THIS AMOUNTS TO
105 MILLION IN 2023 UNDER THE C.
R., PRETTY CLOSE TO WHAT WAS
2022, AND YOU CAN SEE HERE, THE
PIE GRAPHS, THE DISTRIBUTION OF
NONCOMPETING AND COMPETING AND
PAY LINE VERSUS NONPAY LINE
INVESTMENT.
NOW I WANT TO MOVE TO JUST
MENTION BRIEFLY THE ADVANCED
RESEARCH PROJECT AGENCY FOR
HEALTH CALLED ARP A-H, THIS IS A
NEW AGENCY THAT'S SET UP MODELED
ON THE DEFENSE ADVANCE RESEARCH
PROJECT OR DARPA, AND WE ARE
STILL CURRENTLY WAITING FOR A
DIRECTOR TO BE NAMED, THE DEPUTY
DIRECTOR DR. ADAM RUSSELL WAS,A
POINTED AS ACTING AND HIS WORK
TO SET UP THE INFRASTRUCTURE,
ADMINISTRATIVE OFFICES,
FACILITY, I.T., HIRING
ADMINISTRATIVE POSITIONS, BUT
IT'S NOT ENGAGED IN DEVELOPING
THE SCIENCE PROJECTS, AT ARP
A-H, THESE AWAIT THE NAMING OF A
PERMANENT DIRECTOR.
BUT AT OUR LAST COUNCIL, MAY
RECALL WE HAD A VERY SPIRITED
DISCUSSION ABOUT OPEN ACCESS
WHICH IS MAKING PUBLICATIONS
IMMEDIATELY ACCESSIBLE TO THE
PUBLIC AND OF NOTE, THE
PRESIDENT IN AUGUST 25th
SIGNED A NEW POLICY THAT WILL
REQUIRE ALL THE U.S. AGENCIES TO
INSURE IMMEDIATE ACCESS TO
FEDERALLY FUNDED RESEARCH AFTER
THIS PUBLISHED, START NOTHING
2026.
AND SO PEER REVIEW WORK, FROM
THE FEDERAL GRANT RECIPIENTS
MUST BE MADE AVAILABLE IN THE
PUBLIC REPOSITORY WITHOUT DELAY
AFTER PUBLICATION.
NIH IS NOW WORKING WITH THE
STAKEHOLDERS, TO GEP A PLAN AS
WELL OPERATIONALIZE THIS POLICY,
FOR NIH FUNDED RESEARCH AND WE
WILL BE HEARING FROM DR. MICHAEL
LAUER TODAY WHO WILL TALK ABOUT
ANOTHER TOPIC CLOSELY RELATED
AND THAT'S THE DATA MECHANICMENT
SHARING POLICY, AND PERHAPS ALSO
LEAD INTO SOME MENTION ABOUT THE
EQUITABLE ACCESS TO AMERICA'S
RESEARCH POLICY, THE NEW DATA
PLAN REQUIRES A PLAN FOR SHARING
BOTH PUBLISHED AND NONPUBLISHED
DATA AND THIS WILL APPLY TO ALL
APPLICATIONS RECEIVED AFTER
JANUARY 25th, 2023 AND SHY
SILVER BERG FROM THE RESEARCH
QUALITY OFFICE HAS BEEN VERY
ACTIVE IN TRYING TO UNDERSTAND
HOW WE CAN MAKE DATA THAT IS
POSTED, NOT ATTACHED TO THE
PUBLICATION USED BY THE
SCIENTIFIC COMMUNITY AND WILL BE
TALKING ABOUT SOME OF THOSE
IDEAS AT TODAY'S COUNCIL ALONG
WITH DR. LAUER.
SO THANK YOU VERY MUCH FOR THAT
MICHAEL, FOR COMING.
WE WILL WILL TALK A LITTLE BIT
ABOUT THE UNDIAGNOSED DISEASE
NETWORK, THIS IS WAS A COMMON
PROGRAM THAT START INDEED 2013,
AND IT FOCUSES ON BRINGING CASES
OF APPLIED DIAGNOSIS, MANY
CHILDREN, ALSO ADULT WHO IS
RELIES UPON THE CLINICAL
ADVANCES IN CLINICAL GENOMICS TO
MAKE DIAGNOSIS AND THEN TO
UNDERSTAND HOW THESE GENOMIC
VARIANTS GIVE RISE TO DISEASE.
SO IT'S A VERY STRONG RESEARCH
COMPONENT AND ALSO, HAS MADE A
HUGE DIFFERENCE AND BRING SAYING
WHOLE GENOME WIDE SEQUENCING DUE
TO THE DIAGNOSTIC
[INDISCERNIBLE].
NOW IT'S ALL COUNTER POINT
PROJECTS, DO THEY LEAVE THE
COMMON FUND SOURCE OF FUNDING,
AND SOMETHINGMENT AND NINDS,
WHICH HAS BEEN LEADING WITH
NHGRI, SINCE THE BEGINNING, WILL
BE LEADING THE TRANSITION OUT OF
THE COMMON FUND.
AND WE TALKED A LITTLE BIT LAST
TIME ABOUT HOW THE PLAN GOING
FORWARD WILL OCCUR.
WE HAVE RELEASED SOLICITATION
FOR GRANTS FOR THE DATA
MANAGEMENT COORDINATING CENTER
WHICH WILL BE THE HUB FOR THE
UDN OR UDN 2.0 AND THEY WILL
PROVIDE THE INFRASTRUCTURE, DATA
MANAGEMENT AND CLINICAL RESEARCH
SUPPORT FOR A NEW NETWORK OF
CLINICAL SITES THAT WILL
INVESTIGATE PERSONS WITH
UNDIAGNOSED DISORDERS, WE HOPE
TO EXPAND OUT PAST THE GENOMIC
TESTING TO THINGS LIKE,
EXAMINING IMMUNOLOGIC DISORDERS
OR METABOLIC DISORDERS OF THESE
CONDITIONS AS WELL.
WE INSHALLLY AS WE MENTIONED HAD
THE PLAN TO PUT OUT WHAT'S
CALLED AN EXO1 WHICH IS ACCESS
TO THE UDN DATA MANAGEMENT
COORDINATING CENTER FOR CENTERS,
CLINICAL SITES AROUND THE
COUNTRY TO DRAW IN AND TO WORK
IN A THEME SCIENCE MATTER ON
THEIR CASES.
WE DID NOT INITIALLY HAVE THE
EXPECTATION THAT THESE WOULD BE
FUNDED BY NIH BUT THAT MUCH OF
THIS WORK WOULD BE CLINICAL
CARE, BUT WE SEE CONTINUAL
FEEDBACK FROM MANY PATIENTS AND
FAMILIES AT THE SITES THEMSELVES
INDICATE THAT WOULD BE DIFFICULT
TO DO WITHOUT FUNDING.
SO WE'VE NOW HAVE DONE IS PUT
OUT A LIMITED 1 YEAR COMPETITION
TO THE CURRENT SITE THAT WILL
ENABLE THEM TO ENROLL IN NEW
CASES IN THE ENSUING YEAR AND
ESTABLISH THE EFFICIENT
PROCESSES TO WORK WITH THE NEW
DATA MANAGEMENT CENTER, AND
ACTUALLY TO WORK ON EXPANDING
ACCESS TO UNINSURED PARTICIPANT
AND DEVELOP FURTHER
SUSTAINABILITY PLANS GOING
FORWARD.
FY23, WE HOPE THAT WITH THE HELP
OF THE CONGRESS WE WILL BE ABLE
TO EXPAND THE CLINICAL SITE,
FUND THEM TO SOME DEGREE, SO
THAT THEY WILL BE ABLE TO DO THE
RESEARCH COMPONENT AND THE
ACCORD NATION COMPONENT
NECESSARY TO TAKE PART IN THE
UDN AS MAJOR CONTRIBUTORS AND TO
EXPAND THE ACCESS TO SITES THAT
ARE SERVING THE LEADERS OF
COUNTRY OF LOW SOCIOECONOMIC
STATUS TO IMPROVE THE DIVERSITY
OF UDN FOR HOPEFULLY FY2023
PLAN.
>> NOW I'M GOING TO GO AND TALK
ABOUT THE POST ACUTE SEQUELACE
OF COVID, THEY HAVE RELEASED A
STRATEGIC PLAN FOR DEALING WITH
LONG COVID AND THIS INCLUDES
ACTION PLAN AND ALSO SERVICES
SYMPTOMS THAT SUPPORTS LONGER
TERM IMPACT TO COVID-19.
COVID HAS AFFECTED MILLIONS OF
PEOPLE IN THE UNITED STATES AND
WILL LIKELY HAVE A SIGNIFICANT
IMPACT ON THE FUTURE HEALTH OF
THE COUNTRY AND SO THE
GOVERNMENT'S RESPONSE IS TO TRY
TO DEVELOP PLANS TO HELP THOSE
WHO ARE SUFFERING NOW, AND TO
PLAN FOR HOW TO DEAL WITH THESE,
NOW WE TALKED ABOUT IN THE
CURRENT INITIATIVES RESEARCH
SIDE OF THE PLAN AND NIH, WE'VE
SEEN 1.15 BILLION DOLLARS TO
DEVELOP A COMPREHENSIVE RESEARCH
PROGRAM.
AS WE TALKED IN THE PAST, THIS
INCLUDES LONGER TEAM OF COHORT
STUDIES OF THOUSANDS OF
PARTICIPANTS SO THEY CAN BE
MONITORED WITH HOW THEY COVER
OVERTIME AND PIECE APART THOSE
WHO DETERMINE THOSE WHO HAVE A
GOOD RECOVERY VERSUS THOSE WHO
DON'T.
AND ALSO INCLUDES PEOPLE WHO ARE
ALREADY SUFFERING FROM POST
ACUTE SE, EQUELACE, INFECTION,
FROM MONTHS AGO, LONG WITH
CONTROL POPULATIONS AGAIN TO DO
THAT COMPARISON, AND WHAT'S THE
DIFFERENCE BETWEEN THOSE WHO HAD
COVID AND MADE A GREAT RECOVERY,
VERSUS THOSE WHO HAD COVID
INFECTION AND ARE STILL
SUFFERING FROM PERSISTENT
SYMPTOMS.
THERE IS ELECTRONIC HEALTH
RECORD STUDY SYSTEM THAT'S BEEN
SET UP THAT HAS ACCESS TO
60 MILLION RECORDS, ALREADY MADE
SIGNIFICANT OBSERVATIONS ON
INSTANCE AND DIFFERENT TYPES OF
TROUBLES EXHIBITED BY PEOPLE WHO
ARE SUFFERING FROM LONG COVID.
WE FUNDED OR TALKED ABOUT THIS
THE MECHANISTIC STUDIESS, THE
PATHOGENESIS, WHO IS CAUSING IT
FROM PEOPLE WHO HAVE BEEN
INFECTED MONTHS AGO WHO ARE
STILL HAVING TROUBLE AND VERY
CLOSELY TIED TO THIS AND PART OF
THE RECOVERY IS A PATHOLOGY
STUDY, IT'S AN AUTOPSY PROGRAM,
TO DEEPLY INVESTIGATE THE
TISSUES OF THOSE PEOPLE WHO DIED
FOR WHATEVER REASON OR HAD COVID
MONTHS AGO AND TRY TO FIGURE OUT
IF THERE'S SOMETHING THAT CAN BE
SEEN UNDER THE MICROSCOPE THAT
ELUCIDATES WHAT IS THERE AND
WHAT IS THE PERSISTENT SYMPTOM.
THE ENROLLMENT IN THE COHORT
STUDY IS REALLY BEEN QUITE
AMAZING OVER THE LAST FEW
MONTHS, WE'RE NOW OVER 8000
PEOPLE, THIS IS THE LARGEST
PERSON, PATIENT TO PERSON POST
COVID STUDY.
WE HAVE GOOD DISTRIBUTION OF
COURSE OUR DEMOGRAPHICS, OF
COURSE ALSO THE AUTOPSIES
PROGRAM IS PROCEEDING AND HAS
OVER 50 CASES OF PEOPLE WHO HAVE
DIED AND THE TISSUE HAVE BEEN
DONATED FOR TD STUDY TO TRY TO
LOOK UNDER THE MICROSCOPE AND
SEE WHAT IS GOING ON FOR THESE
PERSISTENT SYMPTOMS.
WE ARE NOW ALSO WORKING ON
THRINICAL TRIALS, CONCEPTS AND
MASTER PROTOCOLS, TEST DIFFERENT
SYMPTOMATIC TREATMENTS IN NEEZ
KIND OF MAJOR SYMPTOM CLUSTERS.
THIS IS NOT ALL INCLUSIVE BUT
THE MAJOR PLUSES AS IT TURNS OUT
IN THE LITERATURE AND ALSO IN
RECOVER OR PEOPLE WHO HAVE
TROUBLE WITH AUTONOMIC
DISORDERS, PARTICULARLY THOSE
[INDISCERNIBLE] TACHYCARDIA
SYNDROME, PEOPLE WHO HAVE
TROUBLE WITH EXECUTIVE FUNCTION,
MEMORY, AWMTION CALLED BRAIN
FOG, PEOPLE WHO HAVE TROUBLE
WITH SLEEP, RELATED TO SLEEP OR
FATIGUE WHICH IS THE MOST COMMON
SYMPTOM IN LONG ASHING.
THAT'S ASSOCIATED OF COURSE WITH
MORE EXERCISE INTOLERANT AND
SOME PEOPLE POST EXERTION
MALAISE, A FEATURE THAT'S REALLY
QUITE--SIGNATURE FEATURE HAS
BEEN SEEN IN SOME PEOPLE WITH
POST COVID SYNDROMES AS WELL.
NOW BESIDES THE SYMPTOMATIC
THERAPY, FIND OUT HOW BEST TO
TREAT SYMPTOMS AND PATIENTS ALSO
LOOKING FOR TREATMENTS THAT
COULD POTENTIALLY GET AT THE
UNDERLYING BIOLOGY THAT'S
DRIVING THIS CONDITION WHETHER
IT'S THE VIRAL PERSISTENCE AND
TISSUE IN THE BODY, OR
REGULATION AND TRIGGERED BY
ACUTE REGULATION AND WAS NEVER
REALLY NORMALIZED.
NOW THIS IS VERY, VERY
PRELIMINARY BUT I WANTED TO
THROW IT OUT AS A KIND OF A
POTENTIAL HOME RUN.
SHOULD IT BE VALIDATED, BUT
RECENTLY, THERE HAVE BEEN 2
REPORTS MAYBE OTHERS OF
CIRCULATING EXTRA CELLULAR
VESICLES THAT CONTAIN ANTIGENS
OF SARS COV VIRUS AND THOSE WHO
HAVE POST ACUTE SUPPLY OF COVID.
FOR THESES VESICLES ARE TINY
LIPIDS THAT ARE RELEASED FROM
ALL DIFFERENT CELL TYPES AND
INTO THE BLOOD STREAM AND HAS
BEEN FOUND IS WE CAN SEE IN THE
TOP PANEL THAT THE EQUAL AND
POST ACUTE SEQUELA IN THIS
STUDY, THE RED DOTS ARE HAVE
HARBOR, DESPITE THE PROTEINS IN
THIS YELLOW BOX, OVER MONTHS
AFTER THEIR ACUTE INFECTION,
VERSUS THOSE WHO HAVE
CONVALESCENT, AND GOOD RECOVERY
IN BLUE DO NOT SHOW THIS
CONSISTENT SPEAK PROTEIN IN THE
EXTRA CELLULAR VESICLE.
SOME OF THESE PROTEINS LIKE THE
END PROTEIN IS VERY COMMON AND
THEN GOES AWAY OVER TIME BUT
IT'S A SPIKE PROTEIN THAT WE'RE
WONDERING IS A SIGN OF
CONSISTENT VIRAL INFECTION
SOMEWHERE IN THE BODY OR
CONSISTENTLY RELEASE OF DAMAGED
TISSUE WITH THE VIRAL ANTIGEN
THAT CAN STIMULATE THE IMMUNE
SYSTEM, SO, WHERE WE ARE NOW IS
TRYING TO VALIDATE THESE STUDIES
IN RECOVER COHORT AND VALIDATED
WE'RE PLANNING AN ANTIVIRAL
CLINICAL TRIAL IN PEOPLE WHO
HAVE PICKED OUT BECAUSE THEY
HAVE EVIDENCE OF THIS CONSISTENT
ANTIGENEMIA, SO WE THINK THAT
POST COVID SYNDROME MAY BE A
NUMBER OF DIFFERENT THINGS AND
SOME PEOPLE IT MAY BE AUTOIMMUNE
OR DESREGULATION, SOME PEOPLE IT
MAY BE DO TO VIRAL PERSISTENCE
AND AND IF WE CAN PICK PEOPLE
OUT WE HAVE EYE HIGH CHANCE, WE
HAVE A CHANCE OF WORKING IN THE
EABT VIRAL GROUP AND ALSO WE CAN
USE THE CIRCULATING SPIKE
PROTEIN AS A BIOMARKER OF
SUCCESS AS A DRUG THAT IS
EFFECTIVE AND WILL EXPECT
DESPITE PROTEIN CAN NO LONGER BE
DETECTABLE IN THE CIRCULATING
BLOOD.
SO AGAIN SO THIS IS A LOSS OF
ASSUMPTION WHY DON'T KNOW FOR
SURE IF ANY OF THIS WILL WORK
OUT, WE CAN SEE THE BRIGHT LIGHT
THERE AND THE WHOLE THING
TOGETHER WILL REALLY HELP US TO
ACSALEERATE AND TARGET DOWN ON
TESTING DIFFERENT TREATMENT.
WE ALSO RECENTLY FUNDED
$30 MILLION AND RESEARCH
PROJECTS AND WE ARE LOOKING AT
MANY OTHER POTENTIAL
PATHOPHYSIOLOGICAL MECH NVENLS
UNDERLYING LONG COVID AND POST
ACUTE SEQUELA OF COVID.
YOU CAN SEE ON THE BOX ON THE
LEFT, MANY OF THESE AREISK
BASICALLY NEUROLOGICAL CLINICAL
NEUROSCIENCE PROJECTS LOOKING AT
SLEEP, NEUROVASCULAR SIGNATURES,
P A SC RECOVERY, SPINAL FLUID,
VASCULAR DYSFUNCTION, AUTONOMIC
DYSFUNCTION, BUT ALSO ON THE
RIGHT, CAN YOU SEE A NUMBER OF
CONDITIONS THAT ARE--AFFECT
OTHER BODY SYSTEMS AND THESE ARE
ALSO AND LOOKING AT DIABETES, IT
LOOKS LIKE IT IS INCREASED IN
THOSE PEOPLE WHO HAVE COVID AND
SO THAT IS WHEN METABOLIC
DISORDER THAT'S REALLY BEEN
HIGHLIGHTED AND MOST OF THE
EPIDEMIOLOGICAL STUDIES SO LOOK
AT THE METABOLIC SEQUELA AND I
WANTED TO POINT OUT THAT SOME OF
THE NONHUMAN PRIMATES BECAUSE
THEY REALLY CAN'T REPRODUCE
WHAT'S HAPPENING ON YOUR END AND
RODENTS AND THE INSTANCES AND
NONHUMAN PRIMATES IS REALLY
IMPORTANT AND THAT BRINGS ME TO
THE NEXT POINT I WANTED TO MAKE
THIS IS THAT WE'RE SEEING IN A
SHORTER DENYING HUMAN PRIMATES
AND AVAILABLE FOR RESEARCH IN
THE U.S., THE NEED FOR TESTING
COVID TREATMENTS AND VACCINES
AND TD DEMAND FOR THE MODELS AND
THIS WILL PROBABLY BE OUTPACE
THE RESOURCE SUPPLY IN THE U.S.
AS YOU KNOW NEUROSCIENCE
INCREASINGLY FOR PRIMATES
INCLUDING MONOSETS THAT BRIDGE
OUR UNDERSTANDING OF WHAT WE CAN
DO IN THE MOUSE TO REALLY MAKER
REALLY GOOD HYPOTHESIS TO BE
TESTED ABOUT WHAT HAPPENS IN
HUMANS TO DO THAT BRIDGE, WE
REALLY NEED TO EXAMINE THOSE
KIND OF INTERVENTIONS IN THE
NONHUMAN PRIMATE.
THE BRAIN INITIATIVE AND ALL
THESE RESOURCES AND ALL OF US AS
WE DO WITH ALL OF OUR ANIMALS,
ARE REALLY STRONG FOCUS ON
SHARING THE COMPASSIONATE AND
ETHICAL CARE OF ANIMALS.
THE NATIONAL ACOOED ME OF
SCIENCES ENGINEERING AND
MEDICINE IS CURRENTLY EXAMINING
THE CURRENT ROLE OF FUTURE AND
NEED OF NONHUMAN PRIMATES IN
BIOMEDICAL RESEARCH.
WE LOOK FORWARD TO THEIR REPORT;
I WANT TO SWITCH NOW TO ALS,
STRATEGIC PLANNING.
AS WE MENTIONED IN THE PAST, WE
PUT TOGETHER WORKING GROUP OF
COUNCIL CO-LED BY DR. ROBERT
BROWN, ON OUR COUNCIL AND
DR. RITA SATTLER, AND ALSO AARON
GITLER IS ALSO ON THE ALONG WITH
PERSONS LIVING WITH ALS AND
CAREGIVERS TO TAKE CARE OF THOSE
SUFFERING WITH ALS.
AND THE GOAL OF THE STRATEGIC
PLANNING ACCELERATE RESEARCH ON
THE BIOLOGY BEHIND ALS, AND
QUICKLY AS WE CAN TRANSLATE
FUNDAMENTAL RESEARCH AND
THERAPIES OPTIMIZING CLINICAL
RESEARCH AS YOU KNOW, ALS IS THE
TRAGEC THIS MORNING AND THE
AVERAGE LIFE SPAN AND TRACKING
AND DIFFERENT DIAGNOSES FOR THE
ILLNESS IS ONLY A FEW OF THE
YEAR SO THERE'S A TREMENDOUSLY
END AMOUNT OF URGENCY TO GET AT
THE BOTTOM OF THIS CONDITION AND
TO SEE IF ANY OF THE DISCOVERIES
CAN BE BROUGHT TO THE POINT THAT
WE CAN TEST IT WITH THE ABILITY
TO PREVENT DISABILITY AND DEATH
WITH ALS.
SO IS IT STARTED OUT WITH PUBLIC
INFORMATION WITH OVER 300
RESPONSES, THEY ARE DEVELOPING
DRAFT PILOT PROJECTORRITIES AND
WE APPLY AREAS, BASIC SCIENCE
ALS, TRANSLATIONAL SCIENCE OF
ALS, CLINICAL SCIENCE, QUALITY
OF LIFE, QUALITY OF LIFE FOR
THOSE SUFFERING AND HOW DOES IT
ALL PARTNERSHIP AND
COLLABORATIONS TO ADVANCE THE
SCIENCE MORE QUICKLY.
THIS WILL ALL BE COMING TO A
HEAD AT THE OCTOBER 26th,
27th PUBLIC WORKSHOP, THE
PRIORITIES THAT WE DISCUSSED AND
THE OPPORTUNITIES FOR DISCUSSION
AND COMMENTS, TELL BE A PUBLIC
COMMENT PERIOD, AND IT WILL HELP
US TO REVISE AND HELP US REVISE
THE PRIORITIES TO DEVELOP A
FORMAL SET OF RECOMMENDATIONS
THAT WILL COME TO THE NINDS,
COUNCIL IN FEBRUARY OF 2023, OUR
NEXT MEETING.
SO LOOKING FORWARD TO THIS, I
WANT TO THANK ALL THE FOLK WHO
IS HAVE BEEN WORKING SO HARTD
FOR THE STRATEGIC PLAN.
AS YOU ALSO KNOW AND WE'VE
MENTIONED AT THE LAST COUNCIL,
THAT THE PRESIDENT IN DECEMBER
SIGNED THE ACT FOR ALS BILL AND
PROVISION DIRECTORY RELEVANT TO
NIH INSTRUCTS NIH TO ESTABLISH A
PROGRAM TO FUND GRANTS FOR
SCIENTIFIC RESEARCH ON ALS THAT
UTILIZE EXPABDED ACCESS DATA
FROM INDIVIDUALS THAT ARE NOT
OTHERWISE ELIGIBLE FOR
RANDOMIZED CONTROL TRIALS.
THE PROGRAM IS LIMITED TO TEST
ASSETS THAT ARE CURRENTLY IN
PHASE 3 CLINICAL SITES SPONSORED
BY A SMALL BUSINESS CONCERN.
CONGRESS APPROPRIATED
$25 MILLION TO THE NIH, NINDS
WAS CHARGED WITH SOLICITING THE
GRANTS, REVIEWING THE GRANTS AND
DEVELOPING A FUNDING PLAN THAT
WILL BE DISCUSSED WITH COUNCIL
TOMORROW.
THE BILL WILL ALSO ESTABLISHES A
PUBLIC-PRIVATE PARTNERSHIP WITH
THE FDA AND NIH TO FOCUS ON
ADVANCING REGULATORY SUBSTANCE
AND ACCELERATING SCIENCE
RESEARCH THAT WOULD LEAD TO
EFFECTIVE TREATMENTS FOR ALS,
REALLY LOOKING FORWARD TO
WORKING AND WE HAVE BEEN WORKING
WITH THE FDA TO DEVELOP THE
PUBLIC AND PRIVATE PARTNERSHIP
THAT I THINK WILL BE QUITE
EXCITING AND IMPORTANT.
NEXT I WANT TO JUMP INTO THE
CHRONIC FATIGUE SYNDROME, AS YOU
BE THE COUNCIL WORKING GROUP
DEVELOPED SOME RECOMMENDATIONS
TO NIH AND TO NINDS IN 2019 THAT
WAS BEFORE COVID AND THE
CONDITION REMAINS AS MYSTERIOUS,
ACCEPT AFTER COVID, THERE ARE
NOW MILLIONS OF PEOPLE MORE WITH
MANY OF THE SYMPTOMS OF ME/CFS,
SO I THINK THIS REALLY, COVID
REALLY ESTABLISHED THE
BIOLOGICAL BASIS FOR ANYONE WHO
WAS DOUBTING ABOUT THE CAUSE OF
ME/CFS, AND ALSO THE
INTERSECTION WITH COVID, LONG
COVID SYNDROME AND THE RESEARCH
ARMAMENTARIUM THAT IS BEING
BROUGHT TO UNDERSTAND THAT
CONDITION WE HOPE WILL HAVE
SIGNIFICANT EFFECTS ON NIH'S
RESEARCH ON ME/CFS AS INCLUDING
LONG COVID, BUT NOT COMPLYING TO
LONG COVID BUT WE WOULD LIKE TO
DO THE LARGEST PLANNING PROCESS,
WORKING WITH COUNCIL MEMBERS AND
ME/CFS INVESTIGATORS, ADVOCATES
AND PROVIDERRERS AND INDIVIDUALS
WITH ME-/CFS, WITH THE RESEARCH
STRAY JECTORY TEENINGIC PLAN AND
COME UP WITH A GOAL WHICH OF
WOULD BE THE ME/CFS RESEARCH
TRANSLATIONAL RESEARCH STRATEGIC
PLAN MUCH LIKE WE TALKED ABOUT
A, WELL S.
AND JUST TO WRAP UP, I WANT TO
TALK ABOUT THE LACK OF IN-PERSON
MEETING DUE TO COVID, WE'VE HAD
GREAT WORKSHOPS, BRAIN
INITIATIVE MEETING WAS QUITE
FANTASTIC, OPENING UP TO MORE
PEOPLE THAN WE'VE EVER SEEN
BEFORE AND PAIN AND CONSORTIUM
SYMPOSIA, AND THE INVESTIGATOR
INITTIAIATED MEETING AND
WORKSHOP WITH NCID, AND WE HAVE
A WORKSHOP THAT WAS FANTASTIC A
COUPLE WEEKS AGO, AND
[INDISCERNIBLE] WAS BETTER, I
DON'T KNOW HOW WE'RE GOING TOW
DO IT NEXT YEAR BUT I'M SURE WE
WILL, THAT WAS IN JULY AND
WORKED TOGETHER WITH OVER 70
DIFFERENT DISEASE GROUPS.
SO LOOTS GOING ON, LOTS OF
SCIENTIFIC DISCUSSION, LOTS OF
GREAT RESEARCH BEING PUBLISHED
AND SO WE'RE REALLY PROUD OF OUR
GRANTEES AND OUR STAFF WORKING
THROUGH CONDITIONS THAT HAVE
COME UP US DUE TO THE PANDEMIC.
THE UPCOMING COUNCIL MEETING
THAT WE MENTIONED, WE'RE LOOKING
FOR THE OPTIMAL BALANCE BETWEEN
VIRTUAL HYBRID AND IN-PERSON.
WE HAVE DECIDED THAT THE JANUARY
MEETING OUR NEXT MEETING BECAUSE
OF THE WEATHER ISSUES WILL BE
REMOTE, OUR MAY MEETING WILL BE
IN PERSON AND THEN WE HAVEN'T
DECIDED WHAT WE WANT TO DO IN
SEPTEMBER, WHETHER WE WANT TO GO
HYBRID OR VIRDUAL OR IN PERSON.
--VIRTUAL OR IN PERSON, SO LOOK
FORWARD TO HOW THAT GOES ALONG
AND THIS ISSUE IS, I THINK
FACING ALL ENDEAVORS, ACADEMIC,
SCIENTIFIC, NINDS STAFF IS
PHASING OPTIMIZE HOW TO WORK IN
THIS ENVIRONMENT AS WELL.
SO WITH THEY WANT TO THANK YOU
FOR YOUR ATTENTION.
IT'S BEEN A PLEASURE TO DELIVER
THE WORK THAT WAS SO MANY STAFF
AND GRANTEES, AND HAPPY TO
ANSWER ANY QUESTIONS, THANK YOU
VERY MUCH.
GREAT.
SO HAPPY TO ANSWER QUESTION FIST
ANYBODY HAS ANY, HAPPY TO TRY TO
FIELD THEM.
JUST A SLICE OF WHAT'S HAPPENED
SINCE OUR LAST MEETING BUT
EVERYBODY'S BEEN PRETTY BUSY.
LOTS OF STUFF GOING ON, LOTS OF
OTHER THINGS WE COULD HAVE
TALKED ABOUT AS WELL.
OKAY, I THINK--OH, THERE'S TOM.
>> HELLO WALTER, I'M SURE
EVERYONE'S EAGERLY ANTICIPATING
ARP A-H AND IT LOOKS LIKE FROM
THIS THAT IT'S SORT OF IN
SUSPENDED ANIMATION UNTIL THIS
THERE'S A FULL-TIME DIRECTOR,
WAS THAT THE APPROPRIATE TAKE
HOME?
>> YEAH, THAT'S TRUE, THE THING
ABOUT DARPA IS THAT IT'S THE
SCIENTIFIC PEOPLE THEY HIRE THAT
DETERMINE WHERE THE AGENCY GOES,
SO IT'S NOT LIKE US, WHERE, YOU
KNOW WHERE WE GO DEPENDS A LOT
ON THE GRANTS THAT COME IN, YOU
KNOW IT'S REALLY TOP DOWN, SO
WITHOUT A TOP, YOU KNOW SCIENCE
PERSON THEY REALLY CAN'T MOVE IN
THAT DIRECTION YET.
THE ACTING IS REALLY GOOD
SCIENTIST WITH EXPERIENCE AT
DARPA AND ARP A-H BUT HE'S NOT
IN THE HEALTH SPACE SO WE CAN'T
REALLY HELP ON THE SCIENCE SIDE
BUT HE'S WORKING TO TRY AND
DEVELOP THE INFRASTRUCTURE.
I MEAN A LOT OF PEOPLE ARE
TRYING TO FIGURE OUT HOW TO GET
THEIR TOPICS TAKEN UP BY ARP A-H
AND NOBODY HAS A CLUE YET EXCEPT
WE DO KNOW IS THAT THEY PROBABLY
DON'T WANT IT COMING OUTED OF
NIH, THEY WANT THE IDEAS TO BE
COMING FROM A DIFFERENT
DIRECTION, CERTAINLY THE
CRITICISM HAS BEEN OVER THE LAST
COUPLE OF MONTHS THAT THEY DON'T
WANT THIS TO BE AN EXTENSION OF
THE NIH DIRECTORS, SO PEOPLE ASK
ME TO HELP, I THINK--I THINK IF
I TRY IT'LL BACK FIRE.
TIM?
>> WALTER THIS IS A POLICY
QUESTION RELATED TO JUST
LISTENING TO THE EFFORTS THAT
BOB BROWN AND AARON DID FOR THE
ALS SORT OF STRATEGIC PLANNING.
WELL THE'S ASSUME A YEAR OR NOW
OR WHATEVER, DECISION TO
IMPLEMENT MANY OF THE THINGS
THEY RECOMMENDED, DO YOU
ANTICIPATE, SO IS THAT GOING TO
BE NEW FUNDING EFFORTS SO CARVED
OUT OF EXISTING BUDGETS OR DO
YOU TRY TO GET NEW MONEY TO FUND
THESE NEW INITIATIVES?
YOU'RE ON MUTE.
STILL MUTE.
>> STILL MUTE.
HOW ABOUT NOW?
OKAY.
>> I'M ON THE ROOM MIC SO I
DON'T HAVE CONTROL.
YEAH, SO THAT'S A REALLY GOOD
QUESTION, AND WE FACE THAT IN
ALL DISEASES AND I THINK IF THE
GROUP COMES UP WITH SOME REALLY
SPECTACULAR OPPORTUNITIES THAT
WE NEED TO PUT MONEY ASIDE FOR,
WE WILL HAVE TO CONSIDER THAT IN
TERMS OF MONEY FROM CONGRESS,
YOU KNOW IT'S CERTAINLY HAS
HELPED US A GREAT DEAL IN THE
DEMENTIA SPACE AND THE MONEY
THAT THEY'VE PUT INTO THISAX
PANDEMICKED ACCESS WITH ALS,
WHETHER THEY WOULD EXPAND THAT
FUNDING TO INCLUDE RESEARCH AT
NIH, IS NOT ENTIRELY CLEAR.
THE ODD THING THAT THEY DID WAS
THEY WANT THE FDA TO START A
GRANT PROGRAM, SO, SO IT'S NOT
CLEAR, YOU KNOW BECAUSE NIH IS
USUALLY THE 1 WHO DOES THE
GRANTS, FDA DOES EXPANDED
ACCESS, SO THIS IS A LITTLE BIT
DIFFERENT BUT WE CAN ONLY KEEP
OUR FINGERS CROSSED ON THAT AND
THEN WE HAVE TO, YOU KNOW LIKE
WITH ALL THE OTHER DISEASES, WE
HAVE TO YOU KNOW TRY TO PROTECT
ALL ASPECTS OF OUR SCIENCE BUT
STILL--YOU KNOW TAKE ADVANTAGE
OF OPPORTUNITIES THAT COME
ALONG, YOU KNOW SO THAT WILL
APPLY TO THE FUNDAMENTAL
NEUROSCIENCE, TOO,
THAT--PROBABLY WHY I ASKED.
>> YES, RIGHT.
SO, YEAH, I THINK WE CAN--WE CAN
USUALLY JUGGLE A COUPLE OF
INIAATIVES AT A TIME.
WE CAN NEVER JUGGLE AS MANY AS
PEOPLE WOULD WANT US TO WITHOUT
REALLY THREATENING THE KIND OF
FOUNDATION OF NINDS.
>> RIGHT.
THANKS.
>> VERY STRANGE THAT THERE
AREN'T MORE QUESTIONS, I WILL
PUT PRESSURE ON COUNCIL.
WE HAVE ANOTHER 10 MINUTES
BEFORE THE NEXT SPEAKER, WE
COULD TAKE A BREAK BUT ARE THERE
ANY MORE QUESTIONS?
>> I CAN TALK ABOUT MORE THINGS
IF YOU WANT.
>> WELL, I DON'T KNOW IF WE WANT
TO GO DOWN THAT--THAT COULD TAKE
US WAY FURTHER.
>> IS JOHN HERE?
I SEE HIM,.
>> I'M HERE.
>> SO JOHN, I WAS GOING TO--I
WAS THINKING WHAT DID I FORGET
TO INCLUDE, AND I WAS THINKING
THAT PEDEP WOULD BE SOMETHING
THAT THE COUNCIL WOULD BE
INTERESTED IN HEARING ABOUT, YOU
JUST DID AN ANALYSIS, ANY CHANCE
YOU COULD LIKE SPUR OF THE
MOMENT GIVE A BRIEFING?
>> WELL, THERE'S A CHANCE.
SORRY WE JUST GOT SOMETHING LIKE
A 30 SLIDE PRESENTATION ON THAT
SO THAT MAY BE A LITTLE HARD TO
SUMMARIZE.
>> SO SORRY MY CAMERA IS OUT OF
ACTION RIGHT NOW, JUST SO PEOPLE
KNOW, THE PLAN FOR RISK
PERSPECTIVES IS BEING ROLLED OUT
I THINK RIGHT NOW IN 23 ACTIVE
BRAIN RFAs AND ABOUT
30-SOME-ODD RFAs OUTSIDE OF
BRAIN.
SO IT'S BEING TAKEN UP.
WE DID A VERY PRELIMINARY
ANALYSIS ON THE FIRST 8 RFAs
THAT HAD THE PDP IN THEM, AND
REALLY THIS WAS JUST TO SET UP A
FRAMEWORK IF ARE DOING,AINAL
SIS.
SO THE SHORT VERSION OF THIS IS
WE DON'T HAVE ENOUGH DATA TO
DRAW CONCLUSIONS BUT WE FEEL
CONFIDENT FOR HAVING A GREAT
FRAMEWORK FOR DOING THE
EVALUATION AS THE PDP GETS
ROLLED OUT MORE BROADLY WITHIN
BRAIN AND ACROSS NIH, WE WILL
HAVE A GOOD DATA SET TO LOOK AT.
SO STAY TIEWJED OUR INTEND WAS
TO DO MORE ANALYSIS TO HAVE A
ROBUST DATA SET AND COME BACK TO
THE BRAIN AND MULTICOUNCIL
WORKING GROUP WHICH IS A GROUP
OF THE 10 BRAIN IC COUNCILS AND
ALSO I HOPE TO THIS COUNCIL TO
GIVE YOU AN OWP DATE BUT WE
REALLY NEED TO GATHER A LOT MORE
DATA BEFORE WE CAN COME TO ANY
CONCLUSIONS.
SO STAY TUNED.
I HOPE WHEN THE NEXT COUPLE
MEETINGS, WE WILL HAVE SOMETHING
CREDIBLE TO SHARE WITH YOU BUT
SO FAR, SO GOOD.
I MEAN WIRE NOT SEEING ANY
SURPRISE--WE'RE NOT SEEING ANY
SURPRISES EITHER GOOD OR BAD AT
THIS POINT.
>> THANK YOU.
AND JOHN, CORRECT ME IF I'M
WRONG BUT I LOT OF OTHER ICs
HAVE PICKED THIS UP AND TRIED TO
INCORPORATE INTO THEIR FOISSUE
As AS WELL.
>> YEAH, SO THERE'S QUITE A BIT
OF DISCUSSION ACROSS THE ICs
AND ALSO IN OFFICE EXTRAMURAL
RESEARCH, WE'RE WORKING ON
LANGUAGE THAT WILL BE
STANDARDIZED ACROSS ALL RFAs,
BECAUSE YOU KNOW YOU CAN IMAGINE
THAT THERE'S CERTAIN LINES WE
CANNOT CROSS WITH THIS, WE WANT
TO MAKE SURE WE'RE CONSISTENT IN
DOING THIS RIGHT.
SO IT'S BEING--THERE WILL BE
TUNE UPS BASED ON THE ROLLING
ANALYSIS OF THIS, BUT IT IS
BEING DEPLOYED MORE BROADLY
ACROSS NIH, AND I THINK THAT'S
BOTH THE GREAT THING BUT ALSO A
CHALLENGE TO MAKE SURE IT GETS
DONE AND DONE WELL.
>> OKAY, IF THERE'S NO OTHER
QUESTIONS, WHY DON'T WE JUST
TAKE ABOUT A--LET'S TAKE EXACTLY
A 10 MINUTE BREAK.
WE HAVE A SPEAKER AT 2.
WHY DON'T WE TAKE A QUICK BREAK.
BROOK, DON'T MUTE EVERYBODY
DURING THIS BREAK BECAUSE IF
MIKE LAWER COMES ON, I WANT TO
BE ABLE TO TALK TO HIM.
SO EVERYONE PLEASE JUST MUTE
YOURSELF AND COME BACK IN 9 OR
10 MINUTES.
>>LOOK, SO, WE'RE ABOUT TO HAVE
2 DISCUSSIONS THAT I ACTUALLY
THINK, I SHOULDN'T SAY ACTUALLY,
ALL COUNCIL DISCUSSIONS ARE
INTERESTING, BUT THESE WILL BE
PARTICULARLY INTERESTING, FIRST
SPEAKER IS DR. MICHAEL LAUER,
WHO IS THE DEPUTY DIRECTOR FOR
EXTRAMURAL RESEARCH, HE IS THE
HEAD OF ALL THINGS EXTRAMURAL
AND HE WILL TALK ABOUT THE NEW
NIH DATA MANAGEMENT AND SHARING
POLICY, HE MIGHT EVEN TOUCH A
LITTLE ON THE OSTP DIRECTIVE,
NOD, MIKE IF THAT'S UP TO YOU.
I WASN'T SURE ABOUT THAT.
AND THEN AFTER THAT, I WILL JUST
MENTION THAT WE'RE GOING TO HAVE
A TALK THAT'S SOMEWHAT RELATED
FROM SHAI SILBERBERG, ABOUT THE
IMPORTANCE OF SHARING NEGATIVE
DATA AOPPOSE TO MOST OF THE DATA
THAT'S SHARED SO WITHOUT FURTHER
ADO I WILL TURN IT OVER TO YOUR
SHOW AND YOUR SLIDES, THAT LOOKS
GOOD.
>> GREAT.
>> THANKS FOR COMING.
>> BOB, THANK YOU VERY MUCH.
I'M GLAD, THIS TECHNICAL PROBLEM
TURNED OUT TO BE RELATIVELY
MINOR SO I APPRECIATE THE
INVITATION, TO COME TO OUR
COUNCIL TODAY.
AND I'M GOING TALK ABOUT
IMPLEMENTATION OF OUR DATA
MANAGEMENT AND SHARING POLICY
AND I WANTED TO TAKE THIS
OPPORTUNITY TO POINT OUT THAT
LOTS OF PEOPLE HAVE BEEN WORKING
ON THIS IN PARTICULAR, MY
COLLEAGUES FROM THE OFFICE OF
SCIENCE POLICY, LYRIC JORGENSON,
AND TAUNTON, P A INE, AND THEN
MANY, MANY RPGHTS.
OKAY, SO DATA SHARING IS OF
INTEREST, LITERALLY THEA THE
HIGHEST LEVELS OF GOVERNMENT.
THIS IS SOMETHING THAT PRESIDENT
BIDEN HAS SPOKEN ABOUT, HE ALSO
SPOKE ABOUT QUITE ELOQUENTLY
WHEN HE WAS VICE PRESIDENT, AND
LEADING THE CANCER MOONSHOT
EFFORTS AND IT'S ALSO SOMETHING
WHICH IS EXPLICITLY CALLED OUT
IN THE 21st CENTURY CURES ACT
WHICH HAD A PROVISION, THAT
AUTHORIZED THE NIH DIRECTOR TO
REQUIRE THE SHARING OF
SCIENTIFIC DATA.
IN FACT, DATA POLICIES ARE NOT
NEW, THEY GO WAY BACK BEFORE THE
21st CENTURY CURES ACT, THE
POLICY THAT WE IN PLACE RIGHT
NOW WHICH WILL BE SUPPLANTED IN
JANUARY OF 2023, GOES BACK 20
YEARS TO 2003, ON THIS DATA
SHARING POLICY APPLIED TO AWARDS
THAT ARE MORE THAN 500 K OF
DIRECT COST PER YEAR AND THEN
THERE HAVE BEEN A VARIETY OF
OTHER DATA SHARING POLICIES THAT
HAVE COME ALONG THE WAY,
INCLUDING THE GENOMIC DATA
SHARING POLICY, POLICIES ABOUT
THE SHARING OF CLINICAL TRIAL
INFORMATION, AND THEN A VARIETY
OF DATA SHARING POLICIES RELATED
TO SPECIFIC PROGRAMS OR ICs
AND I'M GUESSING THAT YOU'RE
PROBABLY FAMILIAR WITH AT LEAST
SOME OF THESE.
THIS PARTICULAR POLICY, HAS BEEN
A LONG TIME IN COMING.
IT GOES BACK TO 2016 AND AGAIN,
THAT WAS THE YEAR THAT THE
21st CENTURY CURES ACT WAS
PASSED BY OVERWHELMING
BIPARTISAN, OVERWHELMING BI
PARTIS AN MAJORITIES BY CONGRESS
AND SIGNED BY PRESIDENT OBAMA
AND SINCE THAT TIME THERE HAVE
BEEN A NUMBER OF DRAFTS THAT
HAVE BEEN PUT TOGETHER, SENT OUT
FOR PUBLIC COMMENT, THOSE PUBLIC
COMMENTS HAVE BEEN CONSIDERED
AND THERE'S ALSO BEEN INPUT FROM
VARIOUS PARTS OF GOVERNMENT, THE
FINAL POLICY ITSELF WAS RELEASED
IN OCTOBER OF 2020 AND AT THAT
TIME, WE ANNOUNCED THAT IT WOULD
BE EFFECTIVE FOR APPLICATIONS
COMING IN, IN JANUARY OF 2023.
SO THIS HAS BEEN A LONG PROCESS.
WHY DOES NIH WANT DATA TO BE
SHARED, HOPEFULLY THIS IS FAIRLY
OBVIOUS, BUT IT'S TO ADVANCE
RIGOROUS AND REPRODUCIBLE
RESEARCH AS WELL AS TO PROMOTE
PUBLIC TRUST IN RESEARCH.
AND IF WE THINK ABOUT IT, THE
PICTURE THAT WE HAVE RIGHT NOW,
OF DATA SHARING IS DECIDEDLY
MIXED.
SO LET ME START WITH THE BAD
NEWS.
SO HERE'S AN EXAMPLE OF A
PROBLEMATIC FINDING THIS WAS
FROM THE CENTER OF OPEN SCIENCE
FOR A VERY LARGE SCALE PROJECT
AND SEEING WHETHER OR NOT THEY
COULD REPRODUCE WHAT THEY
CONSIDERED TO BE HIGH IMP PACT,
PRECLINICAL, CANCER BIOLOGY
STUDIES AND THERE WERE NUMEROUS
ARTICLES THAT WERE PUBLISHED.
THIS CAME OUT IN DECEMBER OF
2021.
SO, LITTLE WHILE AGO, AND 1 OF
THE MOST DISTURBING FINDINGS IS
THAT DESPITE CONTACTING THE
AUTHORS OF THE ORIGINAL PAPERS,
THEY WERE UNABLE TO OBTAIN DATA
FOR 68% OF THE EXPERIMENTS, THE
AUTHORS EITHER WERE UNWILLING OR
UNABLE TO PROVIDE THOSE DATA IN
ORDER FOR THE--FOR THESE
PARTICULAR INVESTIGATORS TO
UNDERTAKE THEIR APPLICATION
EFFORTS.
THIS WAS A STUDY THAT WAS
PUBLISHED THIS YEAR, CAME FROM
EUROPE, AND WHAT HAPPENED HERE
IS THAT THESE AUTHORS IDENTIFIED
ABOUT 1800 PAPERS, WHERE THERE
WAS ACTUALLY A DATA AVAILABILITY
STATEMENT.
IT WAS ACTUALLY A STATEMENT
SAYING WE WILL SHARE OUR DATA,
OUR DATA WILL BE AVAILABLE UPON
REQUEST.
THERE'S SOMETHING ALONG THOSE
LINES, AND OVER 90% OF THE CASES
THEY WERE UNABLE TO GET THE
DATA, ONLY 7% OF THE AUTHORS WHO
HAD A DATA AVAILABILITY
STATEMENT ACTUALLY MADE THEIR
DATA AVAILABLE.
SO THAT'S THE BAD NEWS, NOW
THERE IS SOME GOOD NEWS AND THIS
IS A PAPER THAT WAS PUBLISHED BY
MY FORMER COLLEAGUE SEAN CODEY
AT NHLBI AND COLLEAGUES AND WHAT
THEY DID, WAS THEY LOOKED AT
DATA, THAT HAD BEEN DEPOSITED
INTO A REPOSITORY THAT NHLBI
RUNS, AND THEY DEPOSITED DATA ON
100 CLINICAL TRIALS.
THAT'S WHAT WAS LOOKED AT HERE,
THERE'S A LOT MORE DAT THAT'S IN
THERE BUT IN THIS PARTICULAR
CASE, THEY FOCUSED ON 100
CLINICAL TRIALS WHERE INDIVIDUAL
PATIENT LEVEL DATAA WERE
DEPOSITED AND MADE AVAILABLE TO
THE COMMUNITY FOR SHARING AND
WHAT THEY FOUND WAS THAT MOST OF
THE TRIALS, IN FACT, NEARLY 90%
OF THE TRIALS SOMEBODY REQUEST
THE DATA.
AND FURTHER MORE THERE WERE 277
ARTICLES THAT WERE PUBLISHED AND
MANY OF THESE ARTICLES HAD
CITATION VALUES THAT WERE--THAT
WERE QUITE GOOD AND THAT WERE,
LET'S SAY WELL ABOVE THE AVERAGE
FOR NHLBI FUNDED PAPERS AT
LARGE.
NOW, LET ME, I WILL MAKE JUST A
1 QUICK OR 2 QUICK DISCLOSURES
HERE, YOU KNOW 1 IS THAT I
WORKED AT NHLBI ONCE UPON A TIME
AND WE OVERSAW THIS PARTICULAR
DATA REPOSITORY AND THE SECOND
IS THAT BEFORE I CAME TO NIH,
THIS IS NOW, YOU KNOW MAYBE 25
OR SO YEARS AGO, I ACTUALLY USED
SOME OF THESE DATA.
SO THESE DATA WERE AVAILABLE AND
I WAS ABLE TO ACCESS THE DATA
AND WE PUBLISHED SOME PAPERS ON
THE BASIS OF IT.
SO THERE IS SOME GOOD NEWS HERE
WHICH IS THAT DATA SHARING IS
ACTUALLY HAPPENING IN SOME
INSTANCES THIS IS ACTUALLY
YIELDING A SCIENTIFIC RESULTS.
THERE ARE DATA THAT THE PUBLIC
WANTS DATA TO BE SHARED.
THIS IS DATA DONE BY THE PEW
RESEARCH FOUNDATION AND THEY
NOTE THAD PEOPLE ARE MORE LIKELY
TO TRUST SOMETHING IF THEY KNOW
THE DAILY BASIS THEA IS PUBLICLY
AVAILABLE AND THEY'RE LESS
LIKELY TO TRUST SOMETHING IF
IT'S FUNDED BY THE FEDERAL
GOVERNMENT OR BY INDUSTRY.
ALL RIGHT, SO NOW WHAT I WANT TO
DO IS TALK ABOUT THE POLICY
ITSELF.
THERE REALLY ARE 2 PARTS OF THE
POLICY, 1 WILL BE DESCRIBED BY
THE CHAIR, AND THE SECOND PART
OF THE POLICY TO COMPLY WITH THE
PLAN, AND THAT IS EFFECTIVE
JANUARY 25th, 20 TWAI, THAT IS
FOR APPLICATIONS COMING IN,
JANUARY 25th OR 2023 AND
AFTERWARDS.
NOW THERE ARE A LOT OFY DETAILS
AND I AM TALK ABOUT A FEW OF
THEM, 1 IS WE HAVE A DEFINITION
OF WHAT'S ACTUALLY MEANT BY DATA
AND PERHAPS WHAT'S MORE
IMPORTANT IS NOT WHAT'S NOT
MEANT BY DATA SO WHAT'S NOT
MEANT BY DATA ARE LABORATORY
NOTEBOOKS FOR ANALYSIS,
INDIVIDUAL CASE REPORT FORMS,
OBJECTS, BIOSAMPLES, THAT'S NOT
WOWE'RE TALKING ABOUT, WE ARE
FOCUSING ON RESEARCH THAT
GENERATES DATA SO WE KNOW WE
FUND AWARDS THAT DO NOT GENERATE
DATA SUCH AS A RESOURCE AWARDS
OR INSTRUMENTATION AWARDS OR
CONSTRUCTION AWARDS ALSO
TRAINING OR FOLLOW OSHIP,
AWARDS.
IT IS THE POLICIES STATES THAT
DATA NEED TO BE SHARED AT THE
TIME OF PUBLICATION, OR AT THE
END OF AN AWARD FOR DATA THAT
ARE NOT PUBLISHED.
THERE ARE OF COURSE EXCEPTIONS.
THERE ARE--THE GENERAL RULE IS
THAT DATA SHOULD BE--SHOULD BE
SHARED AND THE MORE DATA--THE
IDEA IS TO MAXIMIZE THE AMOUNT
OF DATA SHARING, THAT'S THE
DEFAULT THINKING, ALL DATA HAS
TO BE MANAGED BUT NOT ALL DATA
NEED TO BE SHARED AND WE
UNDERSTAND THAT OF COURSE THERE
ARE LEGITIMATE LIMITATIONS
INCLUDING PROTECTION OF PRIVACY
RIGHTS AND CONFIDENTIALITY AS
WELL AS CERTAIN LAWS, POLICIES
AND REGULATIONS.
A CRITICAL PART OF THE POLICY IS
THAT SCIENTISTS SHOULD BE
THINKING ABOUT MANAGEMENT AND
SHARING OF DATA FROM THE GET-GO.
THIS IS A PROSPECTIVE PROCESS
WHICH OBVIOUSLY WILL EVOLVE OVER
TIME BUT IT'S SOMETHING THAT
SHOULD BE THOUGHT OF AT THE VERY
BEGINNING.
THERE ARE REASONS WHY DATA MAY
NOT BE SHARED, THERE MAY BE
INFORMED CONSENT ISSUES, THERE
MAY BE CONCERNS ABOUT THE PRFACY
OR SAFETY OF RESEARCH
PARTICIPANTS.
THERE MAY BE EXPLICIT LAWS
INCLUDING FOR EXAMPLE, STATE
LOCAL, OR TRIBAL LAWS.
THERE MAY BE RESTRICTIONS DUE TO
AGREEMENTS WITH OTHER PARTIES,
OR THERE MAY BE A VARIETY OF
OTHER PRACTICAL REASONS.
THESE ARE REASONS WHY--THESE ARE
NOT LEGITIMATE REASONS FOR NOT
SHARING DATA, LIKE THE DATA ARE
TOO SMALL, WE DON'T THINK THE
DATA WILL BE WIDELY USED.
WE DON'T THINK THAT THERE'S A
SUITABLE REPOSITORY BECAUSE
THERE IS 1.
AND THERE ARE A VARIETY OF OTHER
CONSIDERATIONS, SBIR, STTR,
THERE IS--IT'S ALLOWED FOR DATA
TO BE WITHHELD FOR 20 YEARS AS
WELL AS TRIBAL SOVEREIGNTY.
ALL RIGHT, NOW, I'M NOT GOING TO
BE ABLE TO TELL YOU EVERYTHING
ABOUT THIS, DURING THIS POINT
TODAY BUT THERE IS A LOT OF
INFORMATION AVAILABLE AND I WANT
TO THANK MY COLLEAGUES,
PARTICULARLY LEE SULLIVAN IN OUR
DIVISION OF COMMUNICATIONS AND
OUTREACH AND HER TEAM, FOR
WORKING WITH OTHERS TO PUT
TOGETHER THIS WEBSITE, THIS
WEBSITE BECAME WAS RELEASED ON
APRIL 5th, IT IS CONSTANTLY
UPDATED, IT'S HERE AT
SHARING.NIH.GOV.
BUT ALSO SHARING POLICIES ABOUT
THE GENOMIC DATA SHARING POLICY
ALSO FOR PEOPLE ON THE OTHER END
LIKE FOR WHEN I WAS A JUNIOR
RESEARCHER, PEOPLE ARE
INTERESTED IN ACCESSING DATA.
SO IF THIS IS THE ONLY THING
THAT HAPPENS TODAY, AND YOU
SPREAD THE WORD ABOUT THIS
WEBSITE THEN I'VE ACCOMPLISHED
SOMETHING USEFUL.
THERE'S QUITE A BIT HERE AND YOU
CAN DIG INTO A VARIETY OF
DIFFERENT TOPICS, THERE IS FOR
EXAMPLE, A WIZARD WHERE YOU CAN
FIGURE OUT WHICH POLICY
SPECIFICALLY APPLY TO YOU AND
THIS IS THE OPENING LINE OF THE
WIZARD AND THEN ADDITION, THERE
ARE LOTS OF RESOURCES.
SO RESOURCES ON DATA SHARING
APPROACHES, ON SELECTING
INAPPROPRIATE DATA REPOSITORY AS
WELL AS LEARNING MORE ABOUT
THESE REPOSITORIES.
THERE'S ALSO BUDGETING AND HOW
DO YOU WRITE OR DO YOU THINK
ABOUT BUDGETING FOR DATA SHARING
AND MANAGING, AS FAR AS HOW DO
YOU WRITE A SPECIFIC PLAN AND WE
HAVE A TEMPLATE FOR EXAMPLE, FOR
HOW SUCH A PLAN SHOULD BE PUT
TOGETHER.
NOW, BACK WHEN THE POLICY WAS
RELEASED, IN OCTOBER OF 2020, WE
IDENTIFIED THEN WHAT WOULD BE
THE APPROPRIATE ELEMENTS OF A
DATA MANAGEMENT AND SHARING PLAN
AND THIS IS--THESE ARE THE
ELEMENTS AT A HIGH LEVEL, WHAT
TYPE OF DATA, RELATED TOOLSOR
SOFTWARE CODE THAT MAY BE
NECESSARY TO ACCESS AND WORK
WITH THE DATA, WHAT STANDARDS
ARE BEING USED, WHERE IS THE
DATA GOING TO BE STORED, WHAT
REPOSITORIES, ARE THERE GOING TO
BE--WHAT ARE THE RULES UNDER
ACCESS, SO SOME DATA MAY BE
READY, SOMEBODY COULD DOWNLOAD
IT AND USE IT AND THERE MAY BE
OTHER KINDS OF DATA WHERE
THERE'S SOME KIND OF CONTROLLED
ACCESS.
AND THEN, WHAT WILL BE THE
OVERSIGHT, YOU KNOW, HOW DO WE
MAKE SURE THAT THE PLAN WILL BE
COMPLIED WITH.
ONE BIG ISSUE, IS WHAT KIND OF
REPOSITORY SHOULD YOU USE.
SO WE STRONGLY ENCOURAGE THE USE
OF ESTABLISHED REPOSITORIES.
NOT A WEBSITE ON THE--NOT AN
EXCEL SPREADSHEET ON THE
LABORATORY WEBSITE BUT FOR
EXAMPLE, IT COULD BE A
REPOSITORY THAT YOUR OWN
INSTITUTION RUNS AND WE KNOW
THAT A NUMBER OF INSTITUTIONS
HAVE PUT TOGETHER SOME
OUTSTANDING REPOSITORIES, NOW IN
SOME CASES, SOME FOAs WILL
SAY, YOU YOU MUST USE XYZ REPOY
AND IT MAY BE AN NIH REPOSITORY
AND THAT MAKES THE DECISION
REALLY VERY SIMPLE.
SO THERE ARE A WIDE VARIETY OF
REPOSITORIES AVAILABLE, ALL OF
THIS INFORMATION IS ON THE
WEBSITE.
THERE ARE NIH SUPPORTED
REPOSITORIES AND SOME OF THEM
ARE SPECIAL REPOSITORIES,
PERHAPS THE 1 THAT'S MOST WELL
KNOWN IS DBGAP WHICH IS RUN BY
THE NATIONAL LIBRARY OF
MEDICINE.
BIOLINK IS THE REPOSITORY THAT
IS USED BY NHLBI, AND THAT WAS
THAT PAPER THEY SHOWED YOU
EARLIER TODAY IS THAT THE
BIOLINK REPOSITORY AND THEN
THERE ARE OTHER REPOSITORIES.
WE POINT OUT THAT A NUMBER OF
INSTITUTIONS AND IN PARTICULAR
INSTITUTIONAL LIBRARIES DON'T
MISS OUT, PUB MED CENTRAL CAN BE
A DATA REPOSITORY BUT THIS IS
ONLY FOR RELATIVELY SMALL DATA
SETS AND THEN THERE ARE A
VARIETY OF GENERALIST
REPOSITORIES AND WE'RE NOT
ENDORSING ANY 1 OF THESE BUT
THESE ARE REPOSITORIES THAT ARE
OUT THERE, AND WIDELY UNUSED.
ALL RIGHT, 1 OF THE BIG
QUESTIONS IS COST.
SO WE DO ALLOW FOR AND IN FACT,
WE EXPLICITLY CALL FOR BUDGETING
FROM DIRECT COSTS, FOR
SUPPORTING DATA MANAGEMENT AND
SHARING.
THERE ARE SOME COSTS THAT ARE
EXPLICITLY ALLOWED LIKE COSTS
FOR CURATING DATA, PRESERVING
IT, SHARING IT, AS WELL AS LOCAL
CONSIDERATIONS.
THIS IS NOT TO BE CONFUSED WITH
CERTAIN KINDS OF INDIRECT COSTS
SO INFRASTRUCTURE COSTS, SO FOR
EXAMPLE, COSTS OF THE LIBRARY,
THAT'S AN INDIRECT COST AND THAT
SHOULD REMAIN THE SAME.
THERE'S BEEN A LOT OF WORK DONE
ON BUDGETING MUCH THE NATIONAL
LIBRARY OF MEDICINE COMMISSIONED
A STUDY BY THE NATIONAL ACADEMY
OF SCIENTISTS THAT IS OUT THERE,
BUDGETING FOR DATA MANAGEMENT
AND SHARING AND WE ANTICIPATE
THAT THERE WILL BE A LOT OF
LEARNING AS WE MOVE ALONG.
ALL RIGHT, NOW THE NEXT THING I
WANT TO TALK ABOUT ARE THE
MECHANICS, HOW WILL THIS
ACTUALLY WORK.
SO, WHEN AN APPLICATION IS
SUBMITTED WITH THE APPLICATION,
THERE NEEDS TO BE AN ATTACHMENT,
WHICH IS THE DATA SHARING AND
MANAGEMENT PLAN.
AND WE HAVE ON OUR WEBSITE, WE
HAVE A, WELL, WHAT'S NOT A DRAFT
BUT LIKELY WILL BE APPROVED BY
OMB, WE HAVE A SUGGESTION FOR
HOW YOU WOULD PUT SUCH A PLAN
TOGETHER, AND THAT SUGGESTION IS
LARGELY BASED ON THE GUIDE
NOTICES THAT WE PUT OUT BACK IN
OCTOBER OF 2020.
SO THAT COMES IN AND THEN THE
OTHER THING THAT COMES IN, IS A
BRIEF BUDGET JUSTIFICATION AND
SO, THERE ARE 2 PARTS TO THIS, 1
IS THE ACTUAL PLAN ITSELF, AND
THAT TYPICALLY WILL BE ABOUT 2
PAGES AND THEN THERE WILL BE A
BRIEF NARRATIVE FOR THE BUDGET
JUSTIFICATION AND THEN WHAT WILL
HAPPEN IS WHEN THE APPLICATION
COMES IN, IF IT GETS TO A POINT
WHERE IT LOOKS LIKE IT'S GOING
TO BE FUNDED, OUR STAFF WILL
ASSESS THE MANAGEMENT AND
SHARING PLAN AND IF IT'S OKAY,
THEN WE'RE GOOD AND THAT PLAN
WILL THEN BE INCORPORATED INTO
THE TERMS AND CONDITIONS OF
AWARD, AND IF--IF THEY HAVE
CONCERNS ABOUT IT OR QUESTIONS
ABOUT IT, THEN THAT WOULD BE A
DISCUSSION THAT WILL HAPPEN
BETWEEN THE NIH STAFF AND THE
POETIC TEBTIAL RECIPIENT.
SO, THIS IS--THIS IS AVAILABLE
NOW, PUBLICLY, THIS IS--WE HAVE
A DRAFT OF WHAT DATA SHARING AND
MANAGEMENT PLAN SHOULD LOOK
LIKE, THE FORMAT HERE IS
OPTIONAL, BUT IT'S FAIRLY
STRAIGHT FORWARD, IT SHOULD BE
NO MORE THAN 2 PAGES IN LENGTH.
WE THEN ALSO HAVE SPECIFIC
PLACES IN OUR ELECTRONIC
INTERFACES WHERE THE PLAN WOULD
BE ATTACHED AND ALSO WHERE THE
BUDGET INFORMATION WOULD BE
ATTACHED SO THAT'S ALL NOW SET
UP, THE PLAN ITSELF ONCE IT IS
APPROVED BECOMES A DETERMINED
CONDITION OF THE AWARD AND LIKE
ALL THERMOGENESISES AND
CONDITIONS OF AWARD--TERMS AND
CONDITIONS OF AWARD WILL BE
CONSIDEREDDA THE TIME OF
PROGRESS REPORTS AND IF THERE'S
A FAILURE TO COMPLY, WE CAN DEAL
WITH IT JUST LIKE WE WOULD DEAL
WITH ANY OTHER NONCOMPLIANCE
CONCERN.
ALL RIGHT, THERE'S A LOT GOING
ON, THERE'S BEEN A LOT GOING ON,
1 IS THAT WE'RE GIVING TALKS
LIKE WHAT I'M DOING RIGHT NOW,
THERE HAVE BEEN WEBINARS,
THERE'S OF COURSE THE WEBPAGE,
WE JUST PUT OUT BY THE WAY, A
GUIDE NOTICE ABOUT HARMONIZATION
WITH THE GENOME DATA SHARING
POLICY, I THINK THAT WENT OUT
ABOUT A WEEK OR SO AGO AND YOU
KNOW WE STRONGLY ENCOURAGE
PEOPLE YOU KNOW TO LOOK AT THE
RESOURCES THAT ARE THERE, ALSO,
TALK TO YOUR LIBRARY, WE KNOW
THAT A NUMBER OF UNIVERSITY
LIBRARIES, AND MEDICAL SCHOOL
LIBRARIES ARE DOING ABSOLUTELY
FANTASTIC WORK IN THIS AREA, SO
THAT MAY BE A VERY VALUABLE
RESOURCE, A LOT OF INTEREST IN
THIS.
THIS IS A PAPER FROM
NORTHWESTERN UNIVERSITY, IT JUST
CAME OUT LAST MONTH, 10 SIMPLE
RULES FOR MAXIMIZING THE
RECOMMENDATIONS OF THE NIH DATA
MANAGEMENT AND SHARING PLAN.
THIS PAPER'S REALLY QUITE GOOD
AND IS WORTH LOOKING AT.
WE'VE HAD A NUMBER OF WEBINARS
AND WE HAVE ANOTHER 1 WHICH IS
COMING UP ON THURSDAY
SEPTEMBER 22nd.
THESE HAVE BEEN WELL ATTENDED,
WE'VE BEEN GETTING A LOT OF VERY
GOOD QUESTIONS AND FEEDBACK, AND
HERE ARE SOME OF THE RESOURCES
THAT ARE AVAILABLE.
SO I HOPE YOU FOUND THIS
INTERESTING AND BOB, I WILL STOP
AT THIS POINT AND TURN THIS BACK
OVER TO YOU.
>> THANKS A LOT MIKE, THAT WAS
GREAT.
COULD WE TAKE DOWN THE SLIDE AND
SEE EACH OTHER.
>> SURE.
THERE YOU GO.
>> THERE ARE QUESTIONS FOR MIKE,
I WILL KICK IT OFF, MIKE, HAVE
YOU DONE ANY--WHAT IS YOUR
THOUGHTS ABOUT THE BUDGETARY
CONSEQUENCES OF THIS?
AS INVESTIGATORS BEGIN TO ASK
FOR MONEY TO BE IN COMPLIANCE
WITH THE POLICY, DO YOU THINK IT
WILL HAVE A SIGNIFICANT
BUDGETARY EFFECT?
I REALIZE THAT'S--HAVE YOU NO
BASIS TO ANSWER THAT QUESTION
BUT I'M CURIOUS.
>> YEAH, WE THINK THAT FOR MOST
WORK, THE BUDGETARY EFFECTS WILL
BE MODEST.
NOW OF COURSE THAT'S VERY
DIFFERENT WHERE WE'RE TALKING
ABOUT, YOU KNOW PEOPLE WHO ARE
PUTTING TOGETHER PEDABYTES OF
DATA AND THEY ALREADY KNOW THAT
AND THEY'RE AWARE OF THAT.
BUT WE THINK FOR MOST STUDIES,
THEY'LL BE REAL BUT THEY'LL BE
RELATIVELY MODEST AND OF COURSE
WHAT WILL HAPPEN IS OVER THE
FIRST 2 OR 3 CYCLES WE WILL GOET
A LOT OF DATA AND THEN WE WILL
BE ABLE TO TALK ABOUT THAT WITH
A BIT MORE AUTHORITY.
>> OKAY, THANKS.
BOB, BOB BROWN?
>> THANK YOU.
NICE PRESENTATION, VERY HELPFUL.
I THINK IT'S VERY GOOD THAT YOU
WILL HAVE PATHWAYS THROUGH THE
WEB SITE THAT WILL FACILITATE
THIS PROCESS, THE ONLY THING
WE'VE RUN INTO WITH PEOPLE
COMMITTED TO THE PROCESS, IS THE
WAI IN WHICH SHARING HAS DONE
OVER THE YEARS, SO THEN WE'VE
GIVEN LARGE SETS OF DATA TO THE
CONSORTIA THEN PROVIDE THE
SHARING INFORMATION AND SOME OF
OUR CONSENTS WHICH WE DID
COMPLETELY IN COMPLIANCE WITH
ALL OF THE APPROPRIATE
REGULATIONS AT THE TIME DO NOT
ADEQUATELY PROVIDE THE
INFORMATION THAT YOU NEED FOR
CONTEMPORARY CONSENTS FOR
SHARING.
SO IT WOULD BE NICE TO HAVE SOME
WAY TO WRESTLE WITH THAT
DEPICTED ON THE WEBSITE.
NYEAH, SO I BELIEVE IT IS--GREAT
QUESTION.
I BELIEVE IT'S ON THE WEBSITE
WHERE NIH HAS DEVELOPED SOME
SUGGESTED CONSENT LANGUAGE FOR
BROAD DATA SHARING, I'M 99% SURE
IT'S THERE, I KNOW WE PUT OUT A
GUIDE NOTICE IN WHICH WE TALKED
ABOUT IT AND THIS IS SOMETHING
THAT JOANY RUDDER WHO IS
DIRECTING NCATS AND MY
COLLEAGUES FROM OSP WORKED ON
FOR PRECISELY WHAT YOU'RE
TALKING ABOUT.
NOW OBVIOUSLY THIS DOESN'T
ADDRESS WHAT HAPPENED 10, 20, 30
YEARS AGO ACCIDENT, BUT YOU KNOW
HOPEFULLY WILL BE HELPFUL
PROSPECTIVELY.
>> THAT IS AN IMPORTANT POINT.
ARNOLD?
>> YES, I APOLOGIZE, I MAY HAVE
MISSED THIS BUT THE ISSUE I'VE
HAD WITH OPEN ACCESS PUBLICATION
IS THAT THE COST THAT THE
JOURNALS, DEMAND FOR OPEN ACCESS
PUBLICATIONS AND SOMETIMES
REQUIREMENTS ARE PUBLISHED WITH
OPEN ACCESS COMES UP AGAINST
THIS REALLY LARGE BARRIER FOR
PUBLICATION.
IS THERE ANY PROGRESS OR ANY
MOVEMENT IN TERMS OF WHAT THE
NIH CAN DO TO HELP MITIGATE
THAT?
>> THAT'S A GREAT QUESTION,
OKAY, SO FIRST OF ALL, OPEN
ACCESS REFERS TO PUBLICATIONS
WHERE THE AUTHOR IS PAY THE
JOURNAL, IN IN CASES, SOME
JOURNALS ARE LIERKS ELIFE OR
[INDISCERNIBLE], THOSE ARE OPEN
ACCESS JOURNALS SO THE ONLY WAY
TO PUBLISH THERE IS YOU PAY THEM
AND THEN THEY PUBLISH THE PAPER.
THEN THERE ARE SOME JOURNALS
THAT ARE BEHIND A FIRE WALL BUT
YOU CAN PAY THEM A FEE, WE KNOW
THE FEES CAN RANGE FROM ANYWHERE
FROM 1000 TO $10,000, I IMAGINE
THAT MAY BE SOME THAT MAY BE
MORE, IN ORDER TO MAKE THE
ARTICLE OPEN.
NIH, NOW WITH CERTAIN
EXCEPTIONS.
THERE ARE SOME FOAs THAT DO
REQUIRE OPEN ACCESS BUT AS A
GENERAL RULE WE DO NOT REQUIRE
OPEN ACCESS, WHAT WE DO REQUIRE
AND PUBLIC ACCESS, AND PUBLIC
ACCESS MEANS THAT THE CONTENTS
OF A MACHINEUE SCRIPT GET
DEPOSITED IN PUB MED CENTRAL AND
GET RELEASED WITHIN 12 MONTHS
AND THAT'S SOMETHING WHERE THERE
IS NO FEE.
YOU KNOW BOB ALLUDED TO THIS NEW
OSTP MEMO, THE OSTP MEMO REFERS
TO PUBLIC ACCESS, NOT OPEN
ACCESS, BUT NONETHELESS, IT IS A
REALLY INTERESTING QUESTION,
THERE ARE SOME PEOPLE AROUND THE
WORLD WHO ARE CALLING FOR ALL
GOVERNMENT GRANTS, ALL
GOVERNMENT FUNDED RESEARCH TO BE
PUBLICIZED THROUGH OPEN ACCESS
AND THE I THINK THE POINT YOU
MAKE IS A REALLY GOOD 1.
YOU KNOW THE COST COULD START TO
GET TO BE VERY REAL.
WE THINK THAT RIGHT NOW, ABOUT
ONE-THIRD OF THE PAPERS THAT ARE
PUBLISHED BY NIH AUTHORS ARE
ALREADY OPEN ACCESS, WE
PUBLISHED--WELL, WE, NIH FUNDED
AUTHORS PUBLISH ABOUT 120,000
PAPERS A YEAR SO THAT MEANS
MAYBE ABOUT 30-40,000 PAPERS A
YEAR ARE ALREADY BEING PUBLISHED
OPEN ACCESS AND YOU KNOW THE
AVERAGE COST MAY BE SOMEWHERE
AROUND $3000, IT'S A LOT OF
MONEY.
>> ACTUALLY MIKE JUST SO YOU
KNOW AT OUR LAST COUNCIL WE
INVITED JOURNAL EDITORS SOMEONE
FROM HOWARD HUGHES AND WE HAD AN
INTENSE DITION CUSHION ABOUT
OPEN ACCESS AS OPPOSED TO CLOSED
ACCESS, JOHN?
>> I APPLAUD THESE EFFORTS,
THANK YOU FOR THE COMMENTS.
IT'S A GREAT DIRECTION AND I'M
PLEASED TO SEE THE AMOUNT OF
THOUGHT THAT'S GENERATED ON WITH
THIS.
I DO THINK THIS WILL HELP WITH
REPRODUCIBILITY BUT THERE IS AN
ISSUE THAT COMES UP WITH DATA
GET USED OVER AND OVER AGAIN.
WHERE YOU'RE RUNNING INTO A RISK
OF TYPE 1S STATISTICAL ERRORS
AND NORMALLY YOU CORRECT THOSE
WITH MULTIPLE COMPARISONS BUT
IT'S PERHAPS GOING TO BE
DIFFICULT TO KNOW HOW OFTEN DATA
SETS HAVE BEEN USED SO I JUST
WONDER IF THIS HAD COME UP IN
DISCUSSION AND WHETHER THERE
WILL BE SOME MECHANISM WHERE AN
INTERESTED PARTY COULD ACTUALLY
KNOW HOW MANY TIMES A DATA SET
HAS BEEN REUSED.
>> YEAH, THAT'S A GREAT QUESTION
AND OF COURSE WE WOULD WANT TO
KNOW HOW OFTEN A DATA SET IS
BEING REUSED BECAUSE THAT'S
PERHAPS A MARKER OF ITS
INFLUENCE, AND YOU KNOW THAT
WILL BE VALUABLE FEEDBACK FOR
EVERYBODY.
SO, YOU KNOW 1 THING THAT WILL
BE VERY MUCH ENCOURAGING IS THAT
A DATA SET WOULD HAVE A DOI,
SOME KIND OF A DIGITAL--DIGITAL
MARKER AND THEN THAT DIGITAL
MARKER COULD THEN BE LINKED TO A
GRANT, COULD BE LINKED TO AN
INSTITUTION, COULD BE LINKED TO
A SCIENTIST, YOU COULD IMAGINE
FOR EXAMPLE, IF YOU HAVE A
PAPER, WITH AN ORCUT ID, THAT'S
THE AUTHOR AND THEN THAT AUTHOR
SAYS I'M USING XYZ DATA AND THAT
DATA HAS A DOI WHICH IS LINKED
TO IT, THEN WE WOULD BE ABLE TO
TRACK THE USE OF DATA JUST LIKE
WE TRACK CITATION OF PAPERS.
AND I THINK YOUR POINTS A REALLY
INTERESTING 1 THAT IF IT GETS
USED OVER AND OVER AGAIN, THE
RISK OF--MULTIPLE COMPARISON
PROBLEM, I THINK, MAY--YOU KNOW
SOMETHING THAT WE WOULD HAVE TO
THINK ABOUT.
>> YEAH, OF COURSE, THE DANGER
IS THAT THE NEGATIVE RESULTS
WON'T GET PUBLISHED THAT WOULD
BE A DISCUSSION FOR A LATER
SESSION HERE.
>> YEAH, ACTUALLY, THE LATER
SESSION WILL OCCUR IN ABOUT 3
MINUTES, BUT TIM WHY DON'T YOU
ASK YOUR QUESTION, TIM IS THE
PERSON WHO SPEARHEADED OUR
DISCUSSION OF OPEN ACCESS IN THE
LASTER COUNCIL AND HE'S FROM
HOWARD HUGHES.
>> MIKE, THANK YOU FOR THE
PRESENTATION, I WANT TO CLARIFY
SO THE WHITE HOUSE MEMO
STIPULATED IT'S ALL ABOUT PUBLIC
ASSET BUT THEY PUT IN THERE
THERE SHOULD BE NO DELAY FOR
ACCESSIBILITY.
SO THE CURRENT POLICY IS A 12
MONTH ACCEPTABLE DELAY, AND THE
JOURNALS ARE OKAY WITH THAT, BUT
WHAT HAPPENS WHEN THIS GETS
SHORTENED TO 0 DAYS DELIAISON
BETWEEN IT PUBLISHING AND
BECOMING OPEN?
>> YEAH THATTA WHAT WE'RE IN THE
MIDDLE OF RIGHT NOW IS WHAT I
WILL SAY IS THAT WE HAVE BEEN
GIVEN A TIMELINE, I THINK WE
HAVE TO TURB IT IN BY FEBRUARY,
WE HAVE TO SUBMIT SOME PLAN TO
OSTP, I THINK THE MOST IMPORTANT
POINT TO MAKE RIGHT NOW IS STILL
VERY MUCH A WORK IN PROGRESS AND
WE'RE AT EARLY STAGES, IS THAT
THERE WILL BE OPPORTUNITY FOR
PUBLIC COMMENT.
>> OKAY.
THANK YOU.
>> OKAY, GINA, LET'S MAKE THIS
THE LAST QUESTION AND THEN MOVE
ON TO SHAI.
>> I KNOW THERE MUST BE
GUIDELINES ON TO WHAT YOU
CONSIDER AN APPROPRIATE
REPOSITORY TO BE BUT IF SOME
RESEARCHER HAS PUT THEIR
REPOSITORY AND ON SOME
COMMERCIAL SITE AND THAT
COMMERCIAL SITE GOES DOWN, WHAT
IS THE--WHAT'S THE THOUGHT
PROCESS THERE?
>> YEAH, THAT'S A GREAT
QUESTION.
SO YES THERE WAS QUITE A BIT OF
WO, THAT WAS DONE ABOUT WHAT
CHARACTERIZES A GOOD REPOSITORY?
AND 1 OF THE DOCUMENTS THAT WAS
RELEASED BACK IN OCTOBER OF 2020
ACTUALLY WENT THROUGH THAT AND
THAT WAS BASED ON A THOROUGH
LITERALLURE REVIEW ON WHAT THOSE
CHARACTERISTICS ARE AND IN
INCLUDED IN THERE IS WHAT
HAPPENS TO DATA IF THE
REPOSITORY GOES BELLY UP, IS
THERE A WAY IN WHICH
SUSTAINABILITY CAN BE ASSURED?
>> OKAY, BEFORE WE--MIKE, YOU
ARE MORE THAN WELCOME TO STAY
FOR SHAI'S TALK WHICH WILL BE
NEXT AND QUITE OVERLAPPING IN
EMERGINGS OF ITS IMPLICATIONS
WITH EVERYTHING YOU JUST SAID
AND THE OSTP DIRECTOR, WALTER DO
YOU WANT TO SAY ANYTHING BEFORE
WE GO OVER TO SHAI?
NO, I WILL JUST SAY THAT AS
EVERYBODY HERE KNOWS WE HAVE
BEEN WORKING REALLY HARD TO
REPRODUCE RIGOR AND ABILITY IN
OUR RESEARCH AND STARTED IN THE
OFFICE OF RESEARCH QUALITY THAT
SHAI LEADS AND SHAI WAS LEADING
OUR ENTRÉE INTO THIS SPACE, I
DON'T KNOW MAYBE 12 YEARS AGO
AND NOW WITH THE DATA SHARING
POLICY, COMING UP, YOU KNOW THE
IDEA OF DATA THAT'S
QUOTE-UNQUOTE POSSIBLE, AND GETS
INTO THE PUBLICATION AND THE
PUBLICATION CAN BE LINKED TO
WHERE THE DATA IS POSTED.
YOU KNOW THAT I THINK IS CLEAR
CUT, YOU KNOW GOING TO BE
BENEFICIAL, BUT, THE PROBLEM
WILL BE FOR DAT THAT DOESN'T
HAVE A LINK TO A PUBLICATION
WHERE THAT MIGHT BE SOME OF THE
MOST FATHOMABLE DATA TO PREVENT
PEOPLE FROM GOING DOWN SIMILAR
RABBIT HOLES AND SO SHAI HAS
BEEN WORKING REALLY HARD ON HOW
TO SOLVE THAT SIDE OF THE
EQUATION FOR THE NIH DATA
POLICY.
SO SHAI?
>> SO I WAS ASKING ABOUT IF YOU
HAD COMMENTS ABOUT WHAT MIKE WAS
SAYING BUT YOU DID FULFILL THAT
AND INTRODUCE SHAI WHICH IS
GREAT, I WILL JUST ADD 1 THING
WHICH IS SHAI SILBERBERG HAS HAD
AN ENORMOUS IMPACT ON THE RIGOR
IMPROVING AT OUR INSTITUTE, NIH,
JOURNALS, ET CETERA, ET CETERA.
SO OVER TO YOU SHAI.
AND HIS OFFICE ALSO INCLUDES
DEVIN CRAWFORD AND A NEW
ADDITION MARIA HOYE,.
>> SO I HAVE TO MOVE MY CAMERA,
I HAVE 2 SCREENS HERE AND SPHR
SOME REASON IT'S INSISTING ON
PRESENT THANKSGIVING ON THE
OTHER SCREEN SO, OKAY, THERE I
AM.
SO GOOD AFTERNOON EVERYONE WHO'S
ON THE EAST COAST AND ALMOST
AFTERNOON IN THE WEST COAST IT'S
GREAT TO SEE YOU ALL ANDIME
HAPPY TO HAVE THE OPPORTUNITY TO
DISCUSS WITH YOU PUBLICATION
BIAS AND WHAT I WILL DO TODAY IS
BRIEFLY DEFINE TUB PUBLICATION
BIAS AND TALK ABOUT
SIGNIFICANCE, CONSEQUENCES OF
PUBLICATION BIAS, WHY IT HAS NOT
BEEN RESOLVED AND HOW
POTENTIALLY IT CAN BE ADDRESSED.
NOW IT'S IMPORTANT TO SAY THAT
WHAT I'M IDEAS I'M GOING TO
PRESENT TODAY, ARE NOT AN NINDS
FUNDING PROPOSAL.
THIS IS JUST A BRAIN CHILD OF
THE OFFICE OF RESEARCH QUALITY
OF HOW WE CAN ADDRESS
PUBLICATION BIAS AND HOW IT HAS
OBVIOUSLY RELEVANCE WITH THE
QUICK TALK WE HEARD FROM
DR. LAUER WHICH WAS HIGHLY
IMPORTANT AND TIMELY.
AND I WANT TO JUST SAY A COUPLE
THINGS ABOUT THIS NIH NOTICE FOR
DATA MANAGEMENT AND SHARING AND
THAT IS THAT, YOU KNOW, AS
WRITTEN IN THE NOTICE, IT'S NOT
ONLY ABOUT DATA THAT IS IN
PUBLISHED PAPERS, BUT ALSO
UNPUBLISHED DATA UNDERLYING
UNPUBLISHED PAPERS INCLUDING
UNPUBLISHED EXPERIMENTS,
INCLUDING EXPERIMENTS THAT DID
NOT SUPPORT THE WORKING
HYPOTHESIS.
AND THERE ARE 2-POINTS I WANT TO
MAKE THAT ARE RELEVANT TO OUR
CONVERSATION TODAY.
THE FIRST IS THAT IN THE
DEFINITION OF SCIENTIFIC DATA,
IT SAYS THAT RECORDING OF
FACTUAL MATERIALS COMMONLY
ACCEPTED IN THE SCIENTIFIC
COMMUNITY AS OF SUFFICIENT
QUALITY TO VALIDATE AND
REPLICATE RESEARCH FINDINGS, IN
OTHER WORDS IT WOULD BE FAIR TO
ASSUME THAT WHEN WE TALK ABOUT
SCIENTIFIC QUALITY, WE ASSUME
THAT THE DATA WAS OBTAINED USING
RIGOROUS METHODS ISSUES THE
DAILY BASIS THEA ISAN OITATED
AND NO DATA IS OMITTED WHICH
COULD JEOPARDIZE THE VALUE OF
UNDERLYING DAILY BASIS THEA.
--DATA.
THE OTHER POINT I WANT TO MAKE
IS THAT SCIENTIFIC RESEARCH
FINDINGS AND DATA ARE NOT 1 AND
THE SAME ASK THEY COULD NOT BE
DUPLICATED.
SO FOR EXAMPLE, IN ADDRESSING
NULL OR NEGATIVE FINDINGS
ACCIDENT IT CLERLY SAYS THAT THE
DATA UNDERLIES THE FINDINGS AND
THAT IS THE FOCUS OF THE NIH
POLICY IS THE DATA THAT
UNDERLIES THE FINDINGS, IN
CONTRAST IN A RECENT GOVERNMENT
ACCOUNTABILITY OFFICE REPORT,
THEY ENCOURAGE THE PUBLICATION
OF NULL RESEARCH RESULTS AND
THAT'S REALLY THE FOCUS OF THE
TALK TODAY.
SO THE NEED FOR DISCUSSING, AND
SHARING RESULTS THAT DO NOT
SUPPORT THE WORKING HYPOTHESIS,
IS BYNY MEANS, NEW.
WE CAN GO BACK TO 1865 TO
CLAUDE, BERNARD WHO SAID IF THE
HYPOTHESIS IS NOT VERIFIED AND
DISAPPEARS, THE FACTS WHICH IT
HAS ENABLED US TO FIND ARE
NONETHELESS ACQUIRED AS
INDESTRECTIBLE MATERIALS FOR
SCIENCE.
I THINK THIS IS A VERY STRONG
STATEMENT.
NOW SINCE THIS STATEMENT WAS
WRITTEN WELL BEFORE THE
STATISTICS REVOLUTION OR
NONHYPOTHESIS OF SIGNIFICANT
TESTING, OBVIOUSLY DID NOT SPEAK
ABOUT NULL RESULTS HERE.
HOWEVER, ALMOST A CENTURY LATER
AT TED STERLING SPECIFICALLY
DISCUSSED THE PERILS OF
STATISTICAL TESTING, HE
RULINNATED ANTIGENSED THAT THERE
IS SOME EVIDENCE IN FIELDS WHERE
STATISTICAL FIELDS OF TESTS ARE
COMMONLY USED, RESEARCH WHICH
YIELDS NONSIGNIFICANT RESULTS IS
NOT PUBLISHED.
AND IT GOES ON TO SAY THAT SUCH
RESEARCH BEING UNKNOWN TO OTHER
INVESTIGATORS, MAY BE REPEATEDLY
INDEPENDENTLY UNTIL EVENTUALLY,
BY CHANCE, A SIGNIFICANT RESULT
OCCURS IN THE AREA OF THE FIRST
KIND S&P PUBLISHED, WHAT THE
SECOND PARAGRAPH BASICALLY SAYS
IS THAT HE'S SPEAKING ABOUT
NONSIGNIFICANT RESULTS THAT ARE
DREW NONSIGNIFICANT RESULTS, NOT
BY CHANCE, OKAY?
AND THE QUESTION IS, YOU KNOW IF
THEY ARE NOT PUBLISHED, THEN HOW
DO WE DEFINE THIS PUBLICATION
BIAS AGAINST NONSIGNIFICANT
RESULTS.
AND THERE HAVE BEEN VARIOUS
VERSIONS FOR THE DEFINITION, I
PERSONALLY LIKE THE DEFINITION,
PUT FORWARD BY KAY DICKERSIN,
WHO SAID THAT PUBLICATION BIAS
IS THE TENDENCYOT PART OF
INVESTIGATORS, REVIEWERS AND
EDITORS TO SUBMIT OR ACCEPT
MANUSCRIPTS FOR PUBLICATION
BASED ON THE DIRECTION OR
STRENGTH OF THE STUDY FINDINGS.
THE REASON I FIND THIS COMPEL
SYSTEM BECAUSE KAY KICKERSIN IMP
IICATES THE REVIEWERS, EDITORS
AND INVESTIGATORRINGS AS
RESPONSIBLE FOR PUBLICATION
BIAS.
SO BEFORE I GO INTO, I WILL
DISCUSS WHO'S RESPONSIBLE FOR
THIS AND HOW, I THINK IT WOULD
BE WORTH WHILE TO LOOK AT THE
EXTENT OF PUBLICATION BIAS.
AND IN THIS REGARD, LET'S LOOK
AT THE WORDS OF BOB ROSENTHALL,
WHO WAS KNOWN FOR THE FILE
DRAWER PROBLEM, WHO SAYS, FORANY
GIVEN RESEARCH AREA 1 CANNOT
TELL HOW MANY STUDY VS BEEN
CONDUCTED BUT NEVER REPORTED.
THE EXTREME VIEW OF THE FILE
DRAWER PROBLEM IS THAT JOURNALS
ARE 50ED WITH 5% OF THE STUDIES
THAT SHOW TYPE 1 ERRORS WHILE
THE FILE DRAWERS ARE FILLED WITH
THE 95% OF THE STUDIES THAT NO
NONSIGNIFICANT RESULTSMENT SO
THIS IS EXTREMELY A SARCASTIC
VIEW ON PUBLICATION BIAS BUT
THERE'S 1 THING THAT'S VERY
CLEAR AND WE COULD ALL AGREE TO
HERE.
AND THAT IS THAT FOR ANY GIVEN
RESEARCH AREA OR CANNOT TELL HOW
MANY STUDIES HAVE BEEN COUNSEL
DUCTED BUT NEVER REPORTED,
RIGHT?
WE CAN KNOW WHAT IS NOT KNOWN
AND THEREFORE TO ESTIMATE THE
EXTENT OF PUBLICATION BIAS,
INVESTIGATORS DEVELOPED ALL
KINDSS OF MODELS THAT ARE BASED
ON VARIOUS ASSUMPTIONS AND
THAT'S WHY I FEEL UNCOMFORTABLE
BECAUSE ONCE YOU HAVE ALL THESE
ASSUMPTIONS, YOU DON'T REALLY
KNOW WHAT IS RIGHT AND WHAT IS
WRONG.
SO I PREFER GOOD OLD IMPERICAL
DATA.
AND I WILL GIVE YOU JUST 2
EXAMPLES OF THAT.
SO IN THIS STUDY BY
[INDISCERNIBLE] AND COLLEAGUES
THEY LOOK AT 2 DATABASES OF
ARTICLES THAT ARE RELATED TO
PROGNOSTIC MARKERS AND FOR BOTH
DATA SETS THEY FOUND THAT LESS
THAN 2% OF THE PAPERS WERE FULLY
NEGATIVE FOR ALL PRESENTED
RESULTS IN THE ABSTRACT.
OKAY?
THAT'S A VERY SMALL NUMBER.
IN THE SECOND PAPER, BY EMILY
[INDISCERNIBLE] AND COLLEAGUES,
THEY LOOK AT 525 UNIQUE
PUBLICATIONS OF ANIMAL STUDIES
OF ACUTE ESCHEMIC STROKE AND
THEY FOUND THAT ONLY 2% REPORTED
SIGNIFICANT EFFECT ON INFARCT
VOLUME AND ONLY 1.2% DID NOT
REPORT AT LEAST 1 SIGNIFICANT
FINDING.
I THINK IT'S INTERESTING HOW
SIMILAR THESE 2 NUMBERS ARE FROM
THESE 2 DIFFERENT STUDIES AND I
THINK IT WOULD BE FAIR TO SAY,
THAT IT'S HIGHLY UNLIKELY THAT
WHEN INVESTIGATOR COMES UP WITH
A HYPOTHESIS, THEY WILL BE
CORRECT 98% OF THE TIME.
SO CLEARLY, PUBLICATION BIAS IS
A SIGNIFICANT ISSUE, IF WE
ASSUME THAT HALF THE TIME
HYPOTHESIS ARE CORRECT, WE WOULD
BE MISSING A HUGE NUMBER OF
STUDIES WHICH BRINGS ME TO AN
EXERCISE OF TRYING TO
GUESSTIMATE OF THE IMPACT OF
PUBLICATION BIAS, SO, WHAT YOU
SEE HERE IS A FIGURE THAT YOU
KNOW I PULLED OUT OF THE NIH
REPORTER, WHICH SHOWS, THE
AMOUNT OF FUNDS THAT NIH SPENT
IN FISCAL YEAR 21 ON RO-1s AND
IN TOTAL IT WAS CLOSE TO 15
BILLION.
NOW IT WOULD BE VERY GENEROUS
AND SAY THAT LET'S ASSUME THAT
80% OF THE TIME WHEN YOU HAVE A
HYPOTHESIS, IT'S CORRECT AND
THAT HALF OF THE OTHER 20% THAT
LED TO NO RESULTS DON'T SEE THE
LIGHT OF DAY.
THEN YOU CAN ESTIMATE THAT
APPROXIMATELY THERE'S 1 AND HALF
BILLION DOLLARS, THE LOSS OF
KNOWLEDGE, JUST DUE TO RO-1
APPLICATIONS, NOW THIS IS
OBVIOUSLY A VERY SIMPLISTIC VIEW
ON THINGS, BUT GENEROSITY OF
ASIDE, IF WE NOW SAY, OKAY, HALF
THE TIME THE HYPOTHESIS IS
CORRECT AND 80% OF THE 1S THAT
DON'T YIELD POSITIVE RESULTS
DON'T SEE THE LIGHT OF DAY, THIS
NUMBER WOULD BE 4 FOLD LARGER.
OKAY?
NOW THIS IS JUST A DIRECT
ESTIMATE OF WHAT THE COST MIGHT
BE, BUT WE ALREADY HEARD FROM
TED STERLING THAT THERE'S
INCORRECT EFFECTS AND ACTUALLY
WALTER MENTIONED THIS AS WELL.
IF OTHER INVESTIGATORS AREN'T
AWARE THAT AN EXPERIMENT WAS
DONE AND YIELDED NO RESULTS,
THEY COULD REPEAT THE SAME
EXPERIMENT.
AND THEN SINCE THEY DON'T
PUBLISH IT, OTHERS WILL REPEAT
THE SAME EXPERIMENT SO THAT ADDS
TO THE EXPENSE OR COST OR LOSS
DUE TO PUBLICATION BY AS.--BIAS,
THERE'S OTHER ELEMENTS AS WELL.
SO TYPICALLY WHEN 1 WANTS TO
ADVANCE TO TRANSLATIONAL AND
CLINICAL STUDIES, YOU TRY TO GET
THE SENSE OF A SIZE OF A
PARTICULAR INTERVENTION AND YOU
GET INFORMATION FROM THE
LITERATE AND YOU ARE COME UP
WITH AN ESTIMATE OF THE EFFECT
SIZE.
NOW IF YOU'RE MISSING ALL THE
NULL RESULTS, USING THIS
INTERVENTION, THEN YOU CAN END
UP WITH THOSE LEADING OR
EXAGGERATED EFFECT SIZES AND
WHAT THIS WILL DO IS WILL LEAD
TO UNDERPOWERED FUTURE STUDIES
BECAUSE WHEN YOU CALCULATE THE
SAMPLE SIZE FOR STUDIES, YOU
TYPICALLY TAKE INTO
CONSIDERATION THE EXPECTED
EFFECT SIZE.
AND SINCE THERE ARE FEW OF THESE
ASKEDS WILL NOW BE UNDERPOWERED,
THEY COULD LEAD TO MISLEADING
RESULTS AND POSSIBLY EVEN TO
BASELESS CLINICAL TRIALS.
SO PUBLICATION BIAS CAN HAVE,
YOU KNOW A PRETTY SIGNIFICANT
EFFECT AND THE QUESTION IS, WHY
PEOPLE DON'T PUBLISH THEIR NULL
RESULTS.
SO WE IN THE OFFICE OF RESEARCH
QUALITY, WE LOOKED INTO THIS, TO
SEE WHAT'S IN THE LITERATURE,
AND COME UP WITH SOME OF OUR OWN
DATA, AND THE FIRST THING WE DID
IS WE LOOKED AT 175 NEUROSCIENCE
JOURNALS THAT DON'T PUBLISH
CLINICAL TRIALS AND ASKED, DO
THEY ENCOURAGE SUBMISSION OF
NULL RESULTS, SO WE WENT OVER
ALL THE DOCK YOU WANTS OF THESE
JOURNALS, AND FOUND THAT ONLY 12
OF THEM SPECIFICALLY RIDICULOUS
MEND OR SUPPORT THE PUBLICATION
OF NULL RESULTS.
WE REACHED OUT TO ALL 12
JOURNALS, TO ASK FOR
INFORMATION, HOW MANY NULL
RESULT PAPERS THEY PUBLISH, WE
GOT RESPONSES ONLY FROM 3 OF
THEM, AND AS YOU CAN SEE, THE
NUMBERS HERE ARE VERY SMALL.
OKAY?
SO THIS IS NOT VERY ENCOURAGING
BUT OBVIOUSLY WE DON'T KNOW HERE
WHY THE NUMBER IS SMALL.
IS IT EDITORIAL CHOICES, THE
DATA WAS NOT HIGH QUALITY OR
PERHAPS REVIEWERS, HAVE SOME
KIND OF BIAS AGAINST NULL
RESULTS.
AND IN THIS REGARD, THERE'S VERY
INTERESTING STUDY THAT WAS
CONDUCTED BY EMERCEON AND
COLLEAGUES, THEY GENERATED 2
VERSIONS OF A WELL DESIGNED
RANDOMIZED CONTROL TRIAL THAT
DIFFERED ONLY IN THE DIRECTION
OF THE FINDINGS OF THE PRINCIPAL
STUDY AND QUHA THEY DID IS THEY
SUBMITTED THIS 22 JOURNALS THAT
WERE PART OF THIS EXPERIMENT AND
THE JOURNALS IN TURN, SUBMITTED
THESE PAPERS THROUGH MANY
INVESTIGATORS FOR REVIEW, AND
RECEIVED RESPONSES FROM 210 OF
THEM.
NOW OBVIOUSLY EACH REVIEWER GOT
ONLY 1 VERSION OF THE PAPER,
RIGHT?
AND WHEN THEY COAALATED ALL THE
DATA, IF THEY RECEIVED PAPERS
THAT HAD A POSITIVE OUTCOME, 90%
OF THEM RECOMMENDED THE PAPER
FOR ACCEPTANCE, WHICH MEANS THIS
WAS--AND WE KNOW IT WAS, WHEN
THEY DESIGNED THIS VERY
CAREFULLY, THE STUDY AND IT WAS
A VERY GOOD PAPER, HOWEVER IF
THE RESULTS WERE NEGATIVE, ONLY
80% SUGGESTED RECOMMENDING THE
ACCEPTANCE OF THIS PAPER.
NOW I FIND THIS VERY DISTURBING
BECAUSE IF YOU THINK OF THOSE
PEOPLE WHO VOLUNTEER TO BE ON
THIS CLINICAL TRIAL, IN THE HOPE
OF ADVANCING MEDICAL TREATMENTS,
AND THEN, ALMOST 1 IN 5
REVIEWERS SAID, OH THIS DATA
DOESN'T NEED TO BE SHARED WITH
THE SCIENTIFIC COMMUNITY BECAUSE
YOU KNOW THE RESULTS ARE
NEGATIVE, OKAY?
IT'S PRETTY DISTURBING.
SO ANYWAY, SO I SHOWED YOU SO
FAR THAT THE JOURNALS DON'T SEEM
TO BE ENCOURAGING THE
SUBMISSION, THERE IS SOME BIAS
TO THE PART OF REVIEWERS, SO,
PERHAPS WHEN ARE NEEDED ARE
JOURNALS THAT ARE DEDICATED TO
NULL RESULTS.
TO OUR KNOWLEDGE THERE HAVE BEEN
8 SUCH JOURNALS SINCE 2002, AND
THEY DID NOT FAIR VERY WELL.
THAT'S THE UNDERSTATEMENT.
IN TOTAL THEY PUBLISHED JUST 470
ARTICLES AND JUST TO PUT THICK
PERSPECTIVE OVER THE SAME TIME
PERIOD, PNAS PUBLISHED ALONE
MORE THAN 65,000 PAPERS.
SO THIS DOES NOT SEEM TO BE AN
AVENUE FOR 1 THAT INVESTIGATORS
USE.
ANOTHER POTENTIAL CONTRIBUTING
FACTOR TO THE FACT THAT
SCIENTISTS DO NOT TONED SUBMIT
THE NULL RESULTS FOR
PUBLICATIONS ARE THE
INSTITUTIONAL POLICIES OF
ENCOURAGING PUBLICATION AND HIGH
PROFILE JOURNALS AND IN THIS
REGARD, PAPER BY [INDISCERNIBLE]
AND COLLEAGUES, THEY DID AN
INTERESTING STUDY, THEY LOOKEDDA
THE DOCUMENTS THAT PROMOTE
REVIEW, PROMOTION AND TENURE AT
50 UNIVERSITIES IN THE U.S. AND
CANADA TO SEE IF THEY USE THE
JOURNAL IMPACT FACTOR IN NEEZ
DOCUMENTS AND HOW THEY USE THEM
AND AS YOU ALREADY KNOW, THE
JOURNAL IMPACT FACTOR IS JUST
THE AVERAGE NUMBER OF TIMES A
PAPER IS CITED IN THE JOURNAL.
AND OBVIOUSLY HAS ABSOLUTELY
NOTHING TO DO WITH THE QUALITY
OR IMPACT OF A SPECIFIC 1 PAPER
IN THAT JOURNAL.
NEVERTHELESS, THEY FOUND THAT 40
INSTITUTIONS, 40% OF THE
INSTITUTIONS MENTIONED THE
JOURNAL IMPACT FACTOR, THE
MAJORITY OF THEM IN A SUPPORTIVE
WAY.
NOW SINCE HIPROFILE JOURNALS ARE
UNLIKELY TO ACCEPT NULL RESULTS,
WE KNOW THAT BECAUSE THAT'S WHAT
MAKES THEM HIGH PROFILE, IN A
SENSE WHAT THIS IS SAYING IS
THAT THE INSTITUTIONS DISCOURAGE
INVESTIGATORS FROM SUBMITTING
NULL RESULTS BECAUSE THEY EXPECT
THEM TO SUBMIT THEM TO HIGH
PROFILE JOURNALS.
SO, IN THIS LANDSCAPE THAT I
JUST DESCRIBED, YOU KNOW, WHAT
ARE THE INVESTIGATORS TO DO?
THEY ARE--YOU KNOW, MANY REASONS
WHY THEY WOULD BE DISCOURAGED
FROM SUBMITTING NULL RESULTS AND
IN FACT IN THIS INTERESTING
STUDY BY FRANCO AND COLLEAGUES
THEY LOOKED AT 221 SURVEY BASED
SOCIAL SCIENCE EXPERIMENTS THAT
WERE FUNDED BY THE NATIONAL
SCIENCE FOUNDATION TO SEE WHAT
THE OUTCOMES OF THE STUDIES
WERE.
AND THEY FOUND THAT IF THE
STUDIES HAD STRONG RESULTS, ONLY
ABOUT 4% OF THE STUDIES WERE NOT
WRITTEN UP, HOWEVER, IF THEY HAD
NULL RESULTS, IT WAS--IT WAS
AROUND 64%, JUST CLEARLY SHOWS
THAT INVESTIGATORS ARE RELUCTANT
TO SUBMIT FULL LENGTH PAPERS ON
NULL RESULTS FOR ALL THOSE
REASONS THAT WE MENTIONED
BEFORE.
BUT THIS RAISES THE POSSIBILITY
THAT MAYBE WHAT IS NEEDED ARE A
PLACE WHERE INVESTIGATORS CAN
SUBMIT SINGLE EXPERIMENTS, YOU
KNOW MICROPUBLICATIONS THAT WILL
BE THE NULL RESULTS.
AND IN THIS REGARD NIH AND THE
NATIONAL LIBRARY OF MEDICINE DID
A VERY INTERESTING EXPERIMENT.
THEY ESTABLISHED AN ONLINE
JOURNAL CALLED A
MICROPUBLICATION BIOLOGY, AND IT
ACCEPTED SINGLE VALIDATED
RESULTS, COULD BE POSITIVE,
NEGATIVE OR REPRODUCIBLE
EXPERIMENTS AND WHAT THEY
REQUIRED IS THAT THE--UNDERLYING
DAILY BASIS AT AND THAT'S
RELATED TO DR. LAUER'S
PRESENTATION WE JUST HEARD, THE
UNDERLYING DATA HAS TO BE SHARED
AS WELL, IT HAS TO BE LINKED TO
THE PUBLICATION AND THEY DID
EVERYTHING POSSIBLE TO MAKE THIS
ATTRACTIVE FOR INVESTIGATORS TO
SUBMIT PUBLICATIONS, SO THEY
MADE THE SUBMISSION FREE, IT
WILL BE PEER REVIEWED WHICH
ALLOWED THEM TO BE CITED ON PUB
MED AND THE DATA WAS CURATED AND
DEPOSITED IN A PLACE PEOPLE CAN
GET TO SO THERE WAS REALLY, YOU
KNOW WHAT COULD BE BETTER THAN
THAT?
WELL AS OF LAST MONTH THERE ARE
ONLY 47 OUT OF THE 504 RESEARCH
PUBLICATIONS ARE SO CALLED
NEGATIVE RESULTS.
ALL RIGHT?
SO THIS PILOT EXPERIMENT
CONDUCTED BY NIH, WAS COMPLETELY
IN LINE WITH THE VIEW THAT WE
HOLD IN THE OFFICE OF RESEARCH
QUALITY THAT THERE'S A MISSING
INGREDIENTS, THAT ALL THESE
THINGS THAT YOU WILL SEE HERE
ARE NOT ENOUGH TO ENCOURAGE
INVESTIGATORS TO SUBMIT MORE
RESULTS AND THEN MISSING
INGREDIENT, IS CHANGING THE
CULTURE OF BIOMEDICAL SCIENCES.
OKAY?
WE HAVE TO CHANGE THE CULTURE,
AND WE THEREFORE THINK THAT NIH
CAN PLAY A SIMPLE ROLE IN
CHANGING THE CULTURE AND TO THIS
END, WE WOULD LIKE TO PROPOSE
AND THIS IS JUST FOR YOUR
INSIGHT AND THOUGHTS, A 3
PRONGED APPROACH.
ONE IS THAT WE HAVE TO INCREASE
THE VALUE OF NULL RESULTS, THE
OTHER IS THAT WE HAVE TO REDUCE
BARRIERS TO PUBLICATION OF NULL
RESULTS, AND THE THIRD IS THAT
WE SOMEHOW NEED TO INCENTIVIZE
BOTH SUBMISSION AND REVIEW OF OF
THESE NULL RESULTS.
AND BECAUSE THESE SLIDE VS BEEN
TRIED BEFORE BECAUSE NONE OF
THEM HAVE TRIED THEM ALTOGETHER
AND CERTAINLY NOT WITH THE
BACKING OF AN ENDORSEMENT FROM
NIH.
SO HOW DO I INCREASE THE VALUE
OF RESULTS, WE WOULD LIKE TO
SUGGEST THAT NIH ESTABLISH A
PLATFORM DEDICATED TO THE
PUBLICATION OF NULL RESULTS,
OKAY?
THIS WOULD SEND A STRONG MESSAGE
FOR COMMUNITY HOW IMPORTANT IS
THIS TO NIH.
NOW THIS PLATFORM CAN BE HOUSED
AT NIH OR BE CONTRACTED OUT,
OKAY, THAT'S NOT MY PAY GRADE OR
YOU TON DECIDE IF NIH DECIDES TO
DO THIS, HOW THEY WANT TO DO IT.
THAT TO ENHANCE THE LEGITIMACY
AND THE QUALITY CONTROL OF
WHAT'S BEING PUBLISHED THERE,
THE NULL RESULTS WILL HAVE TO BE
PEER REVIEWED AND THE PEER
REVIEW WILL BE OF THE
METHODOLOGY ONLY.
SO IT'S ONLY ABOUT WHETHER THE
EXPERIMENT WAS DONE CAREFULLY,
AND WE ALSO PROPOSE THAT THE
UNDERLYING DATA WILL BE LINKED
TO THIS PUBLICATION.
THIS WILL NOT ONLY INCREASE THE
VALUE BUT ALSO WILL GO A LONG
WAY TO SUPPORT THIS NEW NIH
POLICY ABOUT DATA SHARING, AND
MANAGEMENT BECAUSE OTHERWISE,
YOU KNOW THESE RESULTS, IF THEY
WOULD NOT BE PUBLISHED, TELL BE
DIFFICULT TO SEE HOW SCIENTISTS
WOULD ACTUALLY SHARE THIS DATA.
NEXT WE THINK IF WE WANT THIS
PLATFORM TO ACTUALLY BE MINED
FOR ITS CONTENT, TELL BE HAVE TO
BE USER FRIENDLY SEARCH
FUNCTIONS AND EASILY USED TO
TRACK ANYTHING OF VALUE TO THE
SCIENTIFIC COMMUNITY AND
FINALLY, WE THINK THAT IF NIH
REQUIRES INVESTIGATORS TO REPORT
IN THEIR ANNUAL REPORTS AND I'M
PURPOSELY SAYING ANNUAL REPORTS
AND NOT PROGRESS REPORTS BECAUSE
I DON'T LIKE THE NAME PROGRESS
REPORT, BECAUSE THEY IMPLY THAT
REPORTS IS THAT THERE'S
PROGRESS, ABOUT I PREFER TO CALL
THEM ACTIVITY REPORTS, BUT HOW
MANY AND WHICH STUDIES THEY
PUBLISHED AS NULL REPORTS THAT
WOULD SEND A STRONG MESSAGE TO
THE COMMUNITY AND THEN ALLOW
PROGRAM STAFF TO DETERMINE YOU
KNOW HOW TO MOVE FORWARD WITH
THE NEXT YEAR'S FUNDING.
AS FAR AS PUBLIC--REDUCING
BARRIERS, THE FIRST AND FOREMOST
WE THINK THAT THIS PLATFORM
SHOULD BE ONLY ABOUT SINGLE
EXPERIMENTS, NOT ABOUT FULL
PAPERS AND THE ADVANTAGE OF THAT
IS THIS WILL ELIMINATE THE NEED
TO TESTIMONY A STORY, IF YOU
HAVE TO WRITE THIS WHOLE PAPER,
IT'LL BE LIMITED TO JUST A FEW
SENTENCES, WHY THE EXPERIMENT
WAS DONE, VERY DETAILED METHODS
OF HOW THE EXPERIMENT WAS DONE
AND THEN THE ACTUAL RESULT TO
ASSURE THAT FIRST OF ALL IT WILL
BE FREE AND THEN TO ASSURE THAT
IT'LL BE RAPIDLY AND SIMPLY
ACCEPTED ON TO THE PLATFORM AND
REVIEWED RAPIDLY.
WE THINK THERE HAS TO BE
AUTOMATED ADMISSION PROCESS,
WHICH YOU WILL SEE IN A MOMENT
AND ALSO THE SELECTION OF
REVIEWERS WOULD NEED TO BE
AUTOMATED, AND WE HAVE SOME
IDEAS HOW THAT COULD POTENTIALLY
BE DONE BECAUSE THEY'LL ONLY BE
REVIEWING THE METHODS, IT'LL BE
ABOUT EXPERTS IN THE PARTICULAR
METHOD AND POTENTIALLY WE COULD
HAVE INVESTIGATORS SELF-IDENTIFY
AS EXPERTS IN PARTICULAR METHODS
AND THEY COULD BE ATTEMPTED TO
THIS DO THIS, WE HOLD THE VIEW
THAT BECAUSE THEY ONLY LOOKING
AT 1 EXPERIMENT AND ONLY LOOKING
AT IF IT WAS DONE CAREFULLY, THE
BARRIER TO GETTING REVIEWERS TO
REVIEW THIS WOULD BE RELATIVELY
LOW.
AND THERE WILL BE ANOTHER
INCENTIVE IN THIS COMING SLIDE
RIGHT HERE.
SO TO INCENTIVIZE THIS, TELL BE
IMPORTANT THAT THE PUBLICATIONS
ARE INDEXED IN MAJOR BIOMEDICAL
DATABASES.
WE WOULD SUGGEST THAT THE
INVESTIGATORS WOULD INCLUDE
THESE NULL RESULTS IN THEIR
BIOSKETCH AND DURING GRANT
REVIEW WILL BE TAKEN INTO
CONSIDERATION WITH ANY
PRODUCTIVITY WITH AN
INVESTIGATOR.
TO INCENTIVIZE REVIEWERS TO
ACTUALLY REVIEW, 1 COULD
POTENTIALLY MAKE IT POSSIBLE FOR
THE REVIEWS TO GET A DOI AND
ACTUALLY ALSO TO BE INDEXED IN
AS A CITEABLE OBLIGATIONS YECT
AND WE COULD ENCOURAGE REVIEWERS
IN THEIR BIOSKETCH HOW MANY OF
THESE NULL RESULTS THAT I
REVIEWED AND THAT WILL SERVE AS
BROWNIE POINTS AS WELL.
SO TO PUT EVERYTHING IN CONTEXT,
WHAT I JUST TOLD YOU, I SUGGEST
THAT WE LOOK AT THIS NIH
ECOSYSTEM FROM THE PERSPECTIVE
OF INVESTIGATORS IN THE GAME FOR
APPLY FOR NIH GRANTS.
SO WE CAN IMAGINE AN
INVESTIGATOR PERFORMANCE AND
EXPERIMENT, THE EXPERIMENT HE OR
SHE DOES THEIR EXPERIMENT
CAREFULLY AND THE RESULTS TURN
OUT TO BE NULL.
WHAT THEY CAN DO THEN IS TO
SUBMIT THIS TO THIS NEW PLATFORM
THAT YOU'RE PROPOSING, IT'S
FREE, AND IN PARALLEL, THEY WILL
DEPOSIT THE DATA ONCE IT'S WELL
ANNOTATE INDEED A REPOSITORY.
THIS WILL THEN UNDERGO VERY
RAPID AND SIMPLE AUTOMATED
QUALITY CONTROL SIMILAR TO WHAT
HAPPENS IN BIOARCHIVE, WHERE
BASICALLY IT JUST LOOKS AT
TECHNICAL ASPECTS OF THE
SUBMISSION TO SEE THAT IT MEETS
BASIC STANDARDS.
THEN REVIEWERS WILL BE SELECTED
AUTOMATICALLY FROM A DATABASE,
AND WE CAN DISCUSS AFTERWARDS IF
YOU'RE INTERESTED, POTENTIALLY
WHO THESE REVIEWERS MIGHT BE.
IT GOES UNDER, YOU KNOW RAPID
REVIEW, BECAUSE THE REVIEW IS
ONLY ABOUT THE RIGOR AND
METHODS, AND ANALYSIS.
THE TRANSPARENCY OF REPORTING
AND COHERENCE OF THE DOCUMENTS
AND ONCE ACCEPTED, IT RECEIVES A
DOI, AND MAKES IT SEARCHABLE FOR
THE SCIENTIFIC COMMUNITY TO MINE
THIS DATA.
WHAT ARE THE INCENTIVES THAT
THEY WILL GET FROM THIS?
IT'LL BE INDEXED IN PUB MED AS A
PUBLICATION AND ALSO IF IT'S
LISTED IN THE PROGRESS REPORTS,
THIS WORKS IN THEIR FAVOR IN
CONSIDERATION AND CONTINUATION
OF FUNDING, IN PARALLEL, IF THE
INVESTIGATOR PLANS TO SUBMIT A
GRANT FOR HIS OR HER NEXT CYCLE,
THEY CAN LIST THESE IN THEIR
BIOSKETCH AND THEY CAN ALSO LIST
WHEN THEY REVIEWED OTHER SUCH
STUDIES, AND THIS WILL BE TAKEN
INTO CONSIDERATION WHEN
REVIEWING THE INVESTIGATOR IN
THE GRANT APPLICATION.
AND HOPEFULLY THEY RECEIVE THEIR
NEXT FUNDING AND THE CYCLE CAN
REPEAT ITSELF.
SO THAT'S REALLY ALL I HAVE TO
SAY ABOUT THIS ECOSYSTEM AND I
WILL JUST SAY 1 MORE THING AND
THAT IS THIS IS--I'M SHOWING
HERE, FROM THE PERSPECTIVE OF
THE INVESTIGATOR, BUT WE CAN
IMAGINE THAT THERE CAN BE A LOT
OF COLLATERAL BENEFIT AS WELL,
BECAUSE, ONCE THERE'S A CHANGE
IN THE CULTURE THAT NULL RESULTS
ARE VALUED AND APPRECIATED THAT
COULD GO A LONG WAY TO DOING
AWAY WITH THIS CULTURE OF
PUBLISH OR PERISH AND HAVING TO
HAVE POSITIVE RESULTS AND HIGH
PROFILE JOURNALS, SO WITH THAT I
WILL STOP HERE AND THANK YOU FOR
YOUR ATTENTION AND WILL BE GLAD
TO TAKE ANY QUESTIONS YOU MIGHT
HAVE.
>> THANKS A LOT, SHAI, WHY DON'T
WE TAKE THE SLIDES DOWN.
TOM, I SUGGEST EVERYONE READ
GINA'S COMMENTS IN THE CHAT, SHE
WILL BE BACK IN A MINUTE.
TOM?
>> THIS IS GREAT SHAI, THIS IS
SUCH AN IMPORTANT NEW AVENUE,
MOST OF US PROBABLY HAVE HAD
NEGATIVE DATA AND HAVE STRUGGLED
TO FIND PLACES TO PUT IT.
JUST A COUPLE OF QUICK
SUGGESTIONS, THE REVIEWERS
PARTICULARLY WHEN YOU THINK OF
THE ENTIRE BIOMEDICAL RESEARCH
COMMUNITY WILL PROBABLY GET MOST
QUICKLY HUNG UP ON TYPE 2 ERROR.
AND HOW THEY FEEL CONFIDENT
HANDLING RAPIDLY HANDLING THAT
AND SO YOU MIGHT THINK ABOUT A
WAY IN WHICH THAT COULD ALMOST
BE IN A SEMIAUTOMATED WAY DEALT
WITH THE STATISTICAL POWER IS
THERE OR NOT.
YOU KNOW, SO THAT THE REVIEWER
DOESN'T HAVE TO WADE IN AND
THRASH THAT AROUND.
A SECOND IS THAT THERE'S AN
INTERESTING PAPER ON BIOARCHIVES
THAT JUST CAME OUT THAT LOOKED
AT REVIEWERS AND LENGTH OF
REVIEW BY IMPACT FACTOR OF
JOURNAL AND I SUSPECT THAT YOUR
INCENTIVIZATION PROGRAM MAY NOT
BE WORK FOR ALL OF US WHO REVIEW
FOR HIGH IMPACT JOURNALS, SO THE
GREATER THE IMPACT, THE LONGER
THE REVIEW AND THE HIGH IMPACT
JOURNALS PRETTY MUCH EXPECT AS
DETAMED AND LONGER REVIEW,
WHEREAS AS THESE AND WE MIGHT
WANT TO BE RECOGNIZED FOR THAT
MORE THAN THIS.
AND SO THERE MIGHT BE OTHER WAYS
TO THINK ABOUT INCENTIVIZATION,
MAYBE ROLLING SUBMISSIONS, MAYBE
PEOPLE WHO HAVE 5 OF THESE GET
TO SUBMIT JUST LIKE SOMEBODY'S
ON STUDY SECTION, YOU KNOW?
MAYBE THERE ARE OTHER WAYS TO
INCENTIVIZE.
>> GREAT, POINT, TIM.
SHAI, IF YOU WANT TO ANSWER THIS
OF THESE, OTHERWISE, I WOULD
LIKE TO GET AS MUCH FEEDBACK AS
POSSIBLE.
>> THANK YOU SHAI, AND I WANT TO
THANK YOU FOR SINGLE HANDEDLY
PUTTING THE WORD RIGOR IN
CONVERSATION IN THE LAST 10
YEARS AT THE NIH, I THINK WE OWE
GREAT DEBT OF GRATITUDE TO YOU
BUT I WANT TO CHALLENGE A COUPLE
THINGS, I THINK ON PURPOSE WHAT
IS LOST IN THIS IS THE
INTELLECTUAL SIGNIFICANCE OF ANY
FINDING.
WHAT YOU'RE ADVOCATE SUGGEST
THAT HAS TO BE STRIPPED AWAY IN
EVALUATION OF THE PIECE OF DATA
AND I UNDERSTAND TOTALLY THE
MOTIVATION AND POETIC TEBTIAL
IMPACT IT HAS BUT AT THE SAME
TIME, YOU KNOW IF YOU STRIP AWAY
ALL INTELLECTUAL SIGNIFICANCE
YOU COULD SAY LISTEN, I TESTED
THE EFFECT OF THE MICRODOSE,
HOMEIO PATHIC DOSE OF SYNAPSE
FUNCTION AND I HAVE NOTHING, I
WILL PUBLISH THAT, NOW IS THAT
WORTH FILLING UP THESE
REPOSITORIES WITH THAT KIND--I
PICK TODAY RANDOMLY, YOU KNOW
OUT OF A HAT BUT I DO WORRY THAT
BECAUSE THAT'S STRIPPED OF
SIGNATURES HIV CANC, CAN YOU GO
DOWN THAT PATH.
I ALSO WONDER IF 1 HAS
UNDERCOUNTED NEGATIVE RESULTS IN
THE FOLLOWING WAY WHICH IS, YOU
KNOW A TYPICAL GWAS STUDY,
RIGHT?
SHOWS TONS OF NEGATIVE RESULTS,
IT SHOWS 3 THAT DID AND 19,000
TO DIDN'T SO THERE WAS A BUNCH
OF NEGATIVE DATA, RIGHT?
IN MANY DRUG STUDIES YOU MIGHT
COMPARE AN ACTIVE DRUG TO AN
ACTIVE--NEGATIVE RESULT THERE SO
GOOD IS IT SAIDS DO NEED TO HAVE
NEGATIVE THINGS ON THEM
OBVIOUSLY BUT I WOULD SAY IF
THEREYA A COMPELLING ENOUGH
REASON TO DO ANY STUDY, THE
NEGATIVE RESULT SHOULD BE
SIGNIFICANT.
I WORRY ABOUT THE FACT THAT
WE'LL OPEN THE DOOR TO JUST
ANYTHING GOES, YOU DID ANY
EXPERIMENT, DOESN'T MATTER IF IT
WAS A NULL RESULT, YOU KNOW, ALL
COMERS ARE GOOD, NOW I'M JUST
CURIOUS WHAT YOUR PERSPECTIVE IS
ON THAT?
>> WELL, YOU KNOW THAT'S A GREAT
QUESTION, ACTUALLY I HAD A
CONVERSATION WITH BOB ABOUT THIS
JUST--
>> THIS IS EXACTLY--I'M A HUGE
FAN OF IT AND I ASKED HIM THIS
QUESTION YESTERDAY AND WE
DISCUSSED IT FOR AN HOUR
YESTERDAY.
BUT ANYWAY, SORRY.
>> SO YOU'RE OBVIOUSLY RIGHT
THERE. CAN BE REALLY DUMB
EXPERIMENTS THAT PEOPLE DO BUT I
THINK, YOU KNOW EXPERIMENTS THAT
ARE REALLY DUMB WILL DIE OUT.
BUT YOU KNOW THERE ARE PLENTY OF
FULL BLOWN PAPERS OUT THERE THAT
YOU WOULDN'T BOTHER TO READ AND
IF YOU HAVE A GOOD WAY TO SEARCH
THROUGH THIS PLATFORM, THEN YOU
WILL FIND THINGS THAT INTEREST
YOU AND THE ALTERNATIVE IS, NOT
HAVING THE NULL RESULTS, RIGHT?
BECAUSE ONCE YOU START TO SAY,
WELL, IS THIS IMPORTANT OR NOT
IMPORTANT?
WHO'S TO JUDGE THAT, RIGHT?
AND UNFORTUNATELY, PEOPLE THE
WAY THEY JUDGE THINGS, MANY
TIMES IF THE RESULTS ARE
POSITIVE, THEY SAY OH THIS HAS
GOT TO BE IMPORTANT AND OF
COURSE THAT CAN BE
FALSE-POSITIVES ANYWAY, SO, YES,
WE UNDERSTAND, YOU KNOW THAT'S A
POTENTIAL ISSUE, BUT BUT I THINK
THE GAINS ARE MUCH GREATER THAN
THE LOSSES.
>> OKAY, THANKS.
DAVID?
>> HI, SHAI, THIS IS AN
ABSOLUTELY FASCINATING TOPIC OF
CONVERSATION.
WE'VE BEEN THROUGH SOME OF THIS
IN OUR OWN GROUP.
IN FACT, 1 OF THE THINGS IT
TURNED OUT YOU COULD THROW SOME
MONEY AT THE PROBLEM AND IT
HELPED A LOT.
SO 1 OF THE BIG BARRIERS TO
PUBLISHING JUST NEGATIVE RESULTS
WAS THE TIME AND EFFORT IT TOOK
TO ANALYZE THEM AND WRITE THEM
UP, IF YOU ONLY HAVE A CERTAIN
NUMBER OF HOURS IN THE DAY, YOU
WILL CHOOSE TO PRIORITIZE THE
THINGS YOU THINK ARE MOAFORT
IMPORTANT AND THEY COULD BE THE
MOST IMPORTANT NEG5 RESULTS AND
THEY COULD BE THE MOST IMPORTANT
POSITIVE RESULTS BUT YOU
PRIORITIZE YOUR MOST IMPORTANT
RESULTS SO HIRING A PROFESSIONAL
SCIENTIFIC WRITER TO GO THROUGH
AND REALLY HELP WITH THE WRITING
UP ALL OF THEM OR ALL OF THE 1S
THAT WERE VALID OR ALL THE 1S
THAT WERE INTERESTING, STILL
DIDN'T WRITE UP ALL OF THEM BUT
WE WROTE UP MAYBE THE
INTERESTING HALF WHEREAS BEFORE
WE WERE REALLY ONLY FOCUSING ON
THE MOST IMPORTANT QUARTER.
HIRING A SCIENTIFIC WRITERS
COULD REALLY HELP SO THAT'S REAL
MONEY.
I MEAN THE QUESTION THAT AROSE
EARLIER IN THE DIFFERENT TOPIC
ABOUT WHAT WOULD BE THE
BUDGETARY EFFECTS OF THIS
PROPOSAL, I THINK IT WOULD BE
REAL BECAUSE IT WOULD HAVE REAL
ARK DITIONAL STAFF TIME REQUIRED
TO DO THIS, IS IT WORTH IT,
YEAH, IT WOULD BE WORTH IT.
THE OTHER THING TAKING OFF AS A
SCIENTIST AD HOCS AND PUTTING ON
EDITOR IN CHIEF HAT, SO I'M THE
EDITOR IN CHIEF OF NEUROAND
TRAUMA, WE FIND IT HARD TO GET
REVIEWERS TO AAGREE TO REVIEW
NEGATIVE OR NONSIGNIFICANT
STUDIES MUCH HARDER THAN TO DO
THE 1S THAT HAVE A SPECTACULAR
TOP LINE FINDING SO WE HAVE TO
HAVE A BITTER WAY TO INCENTIVIZE
VIEWERS, SO I THINK THE MENINGS
YOU MENTIONED ARE OKAY BUT NOT
GREAT.
SO I THINK WE NEED REALLY
SIGNIFICANT INCENTIVES SO LIKE
FOR EXAMPLE, GIVING IN OUR
JOURNAL WE'RE CONSIDERING THINGS
LOAMACYIC GIVING PEOPLE WHO HAVE
DONE CONSISTENT HIGH QUALITY
ON-TIME REVIEWS FREE OPEN ACCESS
TO A PAPER FOR 1 OF THEIR OWN
PUBLICATIONS OR REDUCED
SUBMISSION CHARGES OR FREE
SUBMISSION CHARGES OR FREE COLOR
FIGURES SO AGAIN PUTTING REAL
MONEY INTO THE--NOT BROWNIE
POINTS BUT LIKE DOLLAR POINTS
INTO THE REWARDS SYSTEM.
SO AND THEN THE THIRD THING IS
THAT WE ALSO HAVE A SPINOFF
JOURNAL CALLED NEUROTRAUMA
REPORTS WHICH IS NOT IN NAME A
NEGATIVE OR NULL RESULTS
JOURNAL, BUT IT DOES MAINLY
PUBLISH A LOT OF NULL REPORTS
AND SO WE ACTUALLY DO PUBLISH
QUITE A NUMBER OF NULL RESULTS.
THEY STILL HAVE TO BE OF GOOD
QUALITY, JUST EXACTLY AS YOU
SAID BUT THEY MAY BE
UNDERCOUNTED IN THE ANALYSIS.
OVER.
>> JOHN?
>> THANKS SHAI, IT'S SUCH AN
IMPORTANT EFFORT, I AGREE WITH
DAVID THE BIGGEST IS TO
INCENTIVIZE INVESTIGATORS TO
PUBLISH THE RESULTS.
I DON'T THINK THE BARRIERS AND
VALUE ARE REALLY ANY QUESTION, I
THINK IT'S CLEAR, THEY NEED TO
BE PUBLISHED, IF I HAD TO GUESS,
I WOULD IMAGINE THE PLACE WHERE
YOU CAN FIND THE MOST NEGATIVE
RESULTS WOULD BE IN CLINICAL
TRIALS BECAUSE THEY'RE
PREREGISTERED AND THEY'RE PUT
TOGETHER CAREFULLY AND YOU KNOW
IT JUST SEEMS LIKE IF YOU ASKED
EVERY INVESTIGATOR FOR A PLAGIN
THE BIOSKETCH WHICH PUBLICATIONS
COME FROM STUDIES THAT WERE
PREREGISTERED, EXPERIMENTS, THAT
IT MIGHT GIVE PEOPLE A LITTLE
MORE INTEREST AND CONCERN ABOUT
WHETHER THEY ARE REPORTING
NEGATIVE RESULTS FROM STUDIES,
THEY'VE DONE AND PREREGISTERED
EXPERIMENTS WILL CERTAINLY
PRODUCE MORE NEGATIVE RESULTS
THAN DISCOVERY EXPERIMENTS.
>> YES, THANK YOU FOR THAT JOHN
AND I--YOU KNOW I FULLY AGREE
THAT, YOU KNOW PREREGISTRATION
OF CLINICAL TRIALS HAS GONE A
LONG WAY TO IDENTIFY, YOU KNOW
FIRST OF ALL IF THINGS WERE DONE
CAREFULLY AND WHERE THE NULL
RESULTS ARE AND SO ON.
FOR DISCOVERY SCIENCE IT'S
UNREALISTIC.
EVEN PROPONENTS, THE PROPONENTS
OF PREREGISTRATION WOULD SAY IT
PROBABLY TAKES ON THE ORDER OF
6-8 MONTHS TO GET IT, YOU KNOW
ACCEPT IT, AND I'M TALKING ABOUT
PREREGISTRATION BUT YOU PUT IT
IN YOUR OWN DATABASE SOMEWHERE
BUT IF YOU WANT TO HAVE THIS
KIND OF GET A STAMP OF APPROVAL
THAT TAKES SEVERAL MONTHS AND IN
PRECLINICAL STUDIES, IT'S, YOU
KNOW UNLIKELY THAT THE PEOPLE
WILL, YOU KNOW, NOT THE WAY
SCIENCE IS DONE, YOU KIND
OF--EXPERIMENTS HAPPEN ON A
WEEKLY BASIS WHERE, OH, WE FOUND
THIS, LET'S DO THIS, YOU WILL
NOT SIT NOW AND WAIT, LET'S
PLAY, LET'S DESIGN THIS
EXPERIMENTS WAIT 6 MONTHS AND
THEN DO THE EXPERIMENT.
SO, AND THE EXPERIMENTS HAVE
SHOWN THAT THIS HAS
ACTUALLY--HASN'T BEEN
SUCCESSFUL, IF YOU LOOK AT
PREREGISTRATION, ALL PLACES DO
PREREGISTRATION, THE VAST
MAJORITY OF STUDIES INVOLVE
HUMAN SUBJECTS AND THE REASON IS
NOT SURPRISING, BECAUSE IT TAKES
TIME BECAUSE YOU HAVE TO HAVE A
CLEAR PROJECT TOGETHER, YOU HAVE
TO GET IRB APPROVAL, ALL THOSE
TAKES TIME SO HAVE YOU TIME TO
PUT IN THE--TO ACTUALLY CREATE
REGISTER.
REGARDING THE REVIEWERS, YOU
KNOW WHICH I AGREE, YOU KNOW
BOTH YOU AND DAVID RAISED THE
ISSUE OF HOW DO YOU INCENTIVIZE
REVIEWERS, I THINK, YOU KNOW
THIS IS JUST AN IDEA, BUT, SINCE
WE ONLY LOOKING AT THE METHOD,
AND IF IT WAS DONE CAREFULLY,
PERHAPS POST DOCS COULD BE, YOU
KNOW GREAT REVIEWERS, BECAUSE
THEY'RE EXPERTS IN THEIR
TECHNIQUE AND FOR THEM, THE
BROWNIE POINTS WILL BE MORE THAN
ENOUGH TO SAY, YOU KNOW WE
ACTUALLY REVIEWED ALL THESE
STUDIES AND HAVE THAT ON THEIR
BIOSKETCH.
IT'S JUST AN IDEA.
>> ANY OTHER COMMENTS?
OKAY, THANKS A LOT SHAI, THAT
WAS INCREDIBLY INTERESTING AND I
KNOW MIKE WAS HERE FOR MOST OF
YOUR PRESENTATION.
BECAUSE WHEN YOU TALK ABOUT IS
AN NIH WIDE EFFORT.
OKAY, WE ACTUALLY ARE AMAZING 3
ENOUGH, EXACTLY ON SCHEDULE.
WHY DON'T WE TAKE A 15 MINUTE
BREAK AND THEN WE WILL TESTIMONY
BACK AND TALK ABOUT THE
ALZHEIMER'S SUMMIT AND OTHER
THINGS.
SO 3:15.
SO THE NEXT SEGMENT -- IS
FROM -- THE POINT PERSON
AHEAD OF THE NANDS'S
ALZHEIMER'S DISEASE PROGRAM
AND HE IS GOING TO BE AN
UPDATE ABOUT THIS AREA
(ADRD). ROD, I ASSUME YOU
WILL INTRODUCE OUR OTHER
SPEAKER WHEN IT COMES TIME
AND WE WILL TALK ABOUT THE
ADRD SUMMIT.
I HOPE I CAPTURED THIS
CORRECTLY.
>>YES, THANK YOU BOB.
LZHEIMER'S DISEASE AND
ALZHEIMER'S DISEASE RELATED
DIMENSIONS ARE IRREVERSIBLE
PROGRESSIVE BRAIN DISEASES
THAT AFFECT MORE THAN 6
MILLION AMERICANS AND THE
NUMBER IS GROWING AS THE
POPULATION AGES. THE MEASURE
HAS AN ENORMOUS IMPACT ON THE
HEALTH, FINANCES AND
WELL-BEING OF INDIVIDUALS,
FAMILIES AND THE SOCIETY. I
ENCOURAGE YOU TO FOLLOW THE
LINK HERE TO SEE A SHORT
PRESENTATION PREPARED BY THE
NADS OFFICE FOR THE ADRD
SUMMIT.
IT'S VERY MOVING AND IT IS
ABOUT THE ONES WHO LIVE WITH
THE CONSEQUENCES OF THIS AND
CONGRESSES CHOSEN TO ACT
INCLUDING THE NATIONAL ACT
THAT OFFERS A HISTORIC
OPPORTUNITY TO ADDRESS
AD/ADRD.
TODAY I WILL PROVIDE AN
OVERVIEW OF THE NISD PROGRAM
AND DOCTOR-- CHIEF OF THE
NEUROLOGY DIVISION THE
MASSACHUSETTS HOSPITAL WILL
PRESENT THE ADRD SUMMIT 2022
REPORT TO THE NANDS COUNCIL.
---
THE NATIONAL ALZHEIMER'S
PROJECT ACT BECAME LAW IN
2011 AND THE NATIONAL PLAN TO
ADDRESS ALZHEIMER'S DISEASE
FIRST ISSUED BY THE
DEPARTMENT OF HEALTH AND
HUMAN SERVICES IN 2012,
INCLUDES DEMENTIAS THAT
SHARE A SIGNIFICANT COGNITIVE
AND PATHOLOGICAL FEATURES
WITH ALZHEIMER'S DISEASE AND
OFTEN COOCCUR WITH THIS AND
THOSE BEINGLEWY BODY
DEMENTIA, FTD, VCID OR
VASCULAR AND MIXED DEMENTIAS.
---
GOAL 1 OF THE NATIONAL PLAN
IS TO PREVENT AND EFFECTIVELY
TREAT ALZHEIMER'S DISEASE,
AND ALZHEIMER'S DISEASE
RELATED DEMENTIAS. THE NIH
RESPONSE TO THE NATIONAL PLAN
LED BY THE NIA INCLUDES
SUMMITS THAT SET SCIENTIFIC
PRIORITIES, MILESTONES AND
IDENTIFY THE MOST URGENT
RESEARCH NEEDED TO MEET GOAL
ONE.
A SUMMIT IS HELD EACH YEAR
ROTATING THROUGH ALZHEIMER'S
DISEASE, ALZHEIMER'S DISEASE
RELATED DEMENTIAS AND
CARE/SERVICES/SUPPORT
RESPECTIVELY.
NIDS LEADS NIH IN ALZHEIMER'S
DISEASE RELATED DEMENTIAS
INCLUDING THE ONE YOU HERE
TODAY AND THENINDDS AND NIA
COLLABORATE CLOSELY ON
FUNDING OPPORTUNITIES AND
SUPPLEMENT PROGRAMS TO EXPAND
THE FIELD, PAY LINES AND
TRIANNUAL SUMMITS AND TODAY I
WANT TO ADD A COUPLE OF WORDS
ABOUT THE NIDS/ADRD SUMMITS.
PART OF THE PROCESS THE
PRESENTATION OF OUTCOMES TO
THE IDEAS COUNCIL. AND THE
RECOMMENDATIONS IS APPROVED
WILL BE SUBMITTED TO THE
DEPARTMENT OF HEALTH AND
HUMAN SERVICES AS HAS BEEN
DONE THE PREVIOUS SUMMER SENT
TO THE NAPA COUNSEL AS
IMPLEMENTATION MILESTONES. IN
ADDITION TO THE NAPA LAW
ANOTHER WAY THE CONGRESS HAS
ACTED IS TO ADD FUNDS
AVAILABLE TO THE NIH FOR
ALZHEIMER'S DISEASE AND
ALZHEIMER'S DISEASE RELATED
DEMENTIAS RESEARCH.
THE NUMBER SHOWN HERE ARE
FROM PUBLIC NIH CATEGORICAL
SPENDING DATA FROM THE
NATIONAL DATABASE AND SINCE
2016 SPENDING HAS INCREASED
MORE THAN FIVEFOLD WITH
SIMILAR INCREASES ACROSS THE
BOARD FOR ALL OF THE AD AND
ADRD DISEASE AND RESEARCH
AREAS.
FUNDING FOR THE NINDS AD/ADRD
PROGRAMS HAS COME FROM CLOSE
COLLABORATION WITH THE
NATIONAL INSTITUTE ON AGING
AND AD FUNDS THAT ARE
TARGETED BY CONGRESS ARE
PROVIDED TO THE ADRD.
YOU CAN SEE HERE IN THE
GRAYBAR THE FUNDS THAT HAVE
BEEN ADDED BECAUSE OF OUR
CLOSE WORK WITH THE NIA.
BOTH INVESTIGATOR INITIATED
RESEARCH AND THE RESEARCH
RESPONSE TO THE ALZHEIMER'S
DISEASE, AND ALZHEIMER'S
DISEASE RELATED DEMENTIA
INITIATIVES, BOTH PLAY
CRITICAL ROLES IN THE WORK
THAT WE DO.
I WANT TO GIVE AN EXAMPLE OF
A RECENT INVESTIGATOR
INITIATIVE.
I WANT TO SHOW A SAMPLE OF
THE RECENT INVESTIGATOR
INITIATED SCIENTIFIC ADVANCE.
THIS 22 STUDY BY KOROBEYNIKOV
AND COLLEGAUES HAS DEVELOPED
-- THE FUSED AND SARCOMA GENE
OR FUS AND COACH AND RNA
BINDING PROTEIN AND CERTAIN
MUTATIONS CAUSE ALS OR FTD.
MOTIVATED BY PROMISING
BIOCHEMICAL AND PHENOTYPIC
RESULTS
IN MODEL-BASED STUDIES AND
CONSISTENT WITH FINDINGS FROM
OTHER STUDIES INCLUDING FROM
TORONIDOL, THIS WILL PUBLISH
THE FIRST IN HUMAN STUDY,
WITH A DRUG ION 363. A
NON-ALLELE SPECIFIC FOS
ANTISENS AND ANTINUCLEOTIDE.
AND YOU CAN SEE ON THE LEFT
PART OF THE SLIDE THE RESULTS
WHICH FOCUSES ON MOTOR
FUNCTIONING DURING TREATMENT.
UNFORTUNATELY THE PATIENT
PASSED AWAY ABOUT A YEAR
LATER, AND AGAIN THIS WAS THE
FIRST IN-HUMAN STUDY. HOWEVER
BIOCHEMICAL AND ANALYSIS
SHOWN HERE HAVE SHOWN THAT
THE TREATMENT DECREASED LEVEL
OF FUS PROTEIN IN THE
PATIENT, AND YOU CAN SEE THAT
IN THIS COLUMN HE COMPARED TO
THE CONTROL COLUMNS.
SO ALSO YOU CAN SEE IT HERE
IN TERMS OF IMMUNO HISTIC
CHEMISTRY. SO THERE IS A LOT
OF WORK LEFT TO DO, BUT
THIS IS AN IMPORTANT STEP
TOWARDS DEVELOPING A
SUCCESSFUL TREATMENT AND IN
TERMS OF RESEARCH AND
PROBABLY RESPONDING TO THE
NINDS FUNDS ANNOUNCED, FUNDS
SINCE 2016 HAVE FACILITATED
MAJOR RESEARCH PROGRAMS THAT
YOU CAN SEE HERE AND INCLUDE
FLACCID PROGRAMS LIKE THE OLD
FTD NATURAL HISTORY STUDY AND
THE NIA CENTER FOR
ALZHEIMER'S RESEARCH OR CAR
HERE. ALL OF THESE PROGRAMS
ARE CONTRIBUTED IMPORTANT
SCIENTIFIC ADVANCES
AND I DON'T HAVE TIME TO DO
JUSTICE TO THE SIGNS
OF THESE TREMENDOUS PROGRAMS
TODAY
BUT I WILL MENTION A FEW.
--- ONE EXAMPLE IS A RECENT
PAPER INVOLVING THE
STRUCTURAL BIOLOGY CONSORTIUM
AND THE LEWI BODY CENTER
WITHOUT WALLS.
---
IN A SERIES OF PAPERS THIS
YEAR, SEVERAL GROUPS
DISCOVERED
AND YOU MISSED ALL THE
PROTEIN THAT IS ASSOCIATED
WITH MULTIPLE AD/ADRD
DIAGNOSIS.
TMEM 106B WAS NOT UNKNOWN AS
A LYSOSOMAL TRANSMEMBRANE
PROTEIN THAT HAVE BEEN
REPORTED PREVIOUSLY AS A RISK
GENE FOR FTD.
AND IF WE LOOK OVER HERE IS
EXPECTED TMEM106B IS
EXPRESSED IN THE BRAINS WITH
NORMAL INDIVIDUALS AND PEOPLE
WITH CONFIRMED FTLD-TDP, PSP
AND DLB.
WHAT IS SURPRISING IS THAT
WHEN THE FTLD/TDP, PSP AND
DLB BRAINS WERE USED TO --
THE MASK SPECTROMETRY TO
DISCOVER THAT TMEM106B IS
BEEN FOLDED SO IT IS BEHAVING
LIKE A CLASSIC MISFORMED
PROTEIN OF DEMENTIA AND
TMEM106B IS PRESENT ACROSS
DIFFERENT AD/ADRD DIAGNOSIS.
AT FIRST IT ACTUALLY SEEMS
QUITE SURPRISING-- AND OF THE
PAST YEARS THERE HAS BEEN
EMPHASIS ON MULTIPLE PATHWAYS
TO DEMENTIA AND INCLUDES RISK
FACTORS SUCH AS FOR LIFESTYLE
ENVIRONMENTS, PSYCHOSOCIAL AS
WELL AS VASCULAR DISEASE AND
CLASSIC MISFOLDED PROTEINS
HERE AND TMEM106B FITS INTO
THIS PICTURE.
AND, IT SORT OF NOT ONLY FITS
IN
THE DRAIN FORCES THAT STILL
EMERGING AND MODERN VIEW OF
ALZHEIMER'S DISEASE AND
ALZHEIMER'S DISEASE RELATED
DIMENSIA AND HOW IT AFFECTS
INDIVIDUALS TO TREAT AND
PREVENT DEMENTIA.
THE TRADITIONAL PERCEPTION OF
RELATIONSHIP BETWEEN CLINICAL
DIAGNOSIS AND PATHOLOGY IS
SHOWN HERE AND YOU SEE THIS
STRAIGHT LINE ARROWS.
I JUST WANT TO MENTION WERE
IDENTIFIED BUT THE REVIEWED
SOMETHING DIFFERENT NOT
MAINSTREAM
AND WHAT WE HAVE LEARNED OVER
TIME IS THAT THIS
TRADITIONAL PERCEPTION IS THE
EXCEPTION
AND NOT THE RULE. THEY ARE
NOW MANY STUDIES THAT HAVE
DEMONSTRATED THIS FROM A
STUDY AT RUSH UNIVERSITY IN
CHICAGO.
THIS STUDY HAS ABOUT 450
PEOPLE FROM COMMUNITY-BASED
POPULATIONS WHO HAD A
CLINICAL DIAGNOSIS OF
PROBABLE ALZHEIMER'S DEMENTIA
DURING LIFE AND DONATED THEIR
BRAINS FOR FOLLOW-UP WITH
PATHOLOGICAL DIAGNOSIS AFTER
THEY PASSED AWAY.
---
ON THE LEFT YOU CAN SEE THAT
THE PROPORTION OF THESE
INDIVIDUALS WHO HAVE ONLY AD
PATHOLOGY IN THE BRAIN IS
VERY SMALL, REPRESENTED BY
THIS THIN WEDGE RIGHT HERE.
ON THE RIGHT WHICH COMBINES
ALL OF THE GROUPS THAT HAD AD
PATHOLOGY WITH AT LEAST ONE
OTHER PATHOLOGY, IT IS VERY
CLEAR THAT MIXED PATHOLOGIES
DOMINATE.
WHAT IS ALSO INTERESTING IS
THAT A SIGNIFICANT PROPORTION
OF THOSE CLINICALLY DIAGNOSED
WITH PROBABLE ALZHEIMER'S
DEMENTIA DON'T HAVE AD
PATHOLOGY AT ALL.
AND YOU CAN SEE SOME OF THOSE
IN THESE TWO WEDGES HERE, FOR
OD+VASCULAR AND VASCULAR
ONLY.
---
WE CANNOT RELIABLY KNOW THE
PATHOLOGIST PRESENT BASED ON
CLINICAL DIAGNOSIS ALONE; WHY
DOES THIS MATTER?
IT MATTERS BECAUSE WE MUST
TREAT INDIVIDUALS WITH
INCREASED RECOGNITION THAT
MORE THAN ONE OF THESE
PROCESSES TYPICALLY PRESENT
IN A PERSON'S BRAIN CAN BE
MOVED TO HEALTH TREATMENTS
AND RECOGNITION OF THOSE
PROCESSES THAT ARE RELEVANT
IN INDIVIDUAL'S BRAIN --
MAKES US TURN TO BIOMARKERS.
---
AN EXAMPLE.
THERE ARE A NUMBER OF
PROGRAMS SUPPORTED BY NINDS
ON BIOMARKERS AND MARK --
VCID WAS ESTABLISHED BY
INITIATIVES DESIGNED TO
CREATE A NATIONAL CONSORTIUM
OF SCIENTIFIC AND
EXPERIMENTAL INFRASTRUCTURE
TO DEVELOP VIA A MULTISITE
TESTING AN INSTRUMENTAL
VIOLATION AND ULTIMATELY
CLINICAL VALIDATION OF VCID
MARKERS INCLUDING,
ARTERIOSCLEROSIS,
CEREBROVASCULAR REACTIVITY,
MRI FREE WATER, PEAKED
SKELETONIZED
D MEAN DIFFUSIVITYAND ALL OF
HIS CLINICAL VALIDATION
STUDIES ARE APPROPRIATELY
POWERED FOR LONG TERM TRIALS
INCLUDING BLACK AND HISPANIC
POPULATIONS.
OTHER NINDS/ADRD PROGRAMS
THAT ADDRESS HEALTH EQUITY
INCLUDE THE DETECTCID
PROGRAM, AN EFFORT TO IMPROVE
THE QUALITY AND FREQUENCY OF
COGNITIVE IMPAIRMENT IN
EVERYDAY CLINICAL SETTINGS.
DETECTCID HAS ENTERED ITS
SECOND PHASE IN PRAGMATIC
CLINICAL TRIALS ARE UNDERWAY.
THE REGARD STUDIES HAS BEEN A
LONG-TERM STUDY THAT IN
ADDITION TO STROKE HAS
INCLUDED A FACTOR ON VASCULAR
CONTRIBUTIONS TO COGNITIVE
IMPAIRMENT VCID.
WE HAVE A RESEARCH SUPPLEMENT
PROGRAM TO PROMOTE DIVERSITY
IN HEALTH RELATED RESEARCH.
I ALREADY MENTIONED MARK
VCID.
BUT DISCOVERY TO UNDERSTAND
HOW STROKING COMORBIDITIES
LEAD TO DEMENTIA AS WELL AS
DIVERSE VCID REGARDING THE
CLINICAL SIGNIFICANCE OF
INCIDENTAL WHITE MATTER
LESIONS FOR DEMENTIA. ALL OF
THESE PROGRAMS REQUIRE THAT
DIVERSITY BE EXPLICITLY
CONSIDERED BOTH IN THE RFA
AND IN THE MILESTONES OF
THESE PROGRAMS.
---
THE MIND YOUR RISKS CAMPAIGN
IS ANOTHER WONDERFUL WAY THAT
THE NIDS PRIORITIZES AN
ACTION HEALTH EQUITY AND THE
IMPORTANCE OF CONTROLLING
BLOOD PRESSURE TO PREVENT
BOTH STROKE AND DEMENTIA.
THIS AGAIN WAS CREATED BY THE
NINDS ONCE OFFICE.
I ENCOURAGE YOU TO LOOK AT IT
AND I WANT TO TAKE A MOMENT
TO TALK ABOUT THE NINDS
FUNDING INITIATIVE THAT IS
ALREADY OPEN OR PLAN FOR
FISCAL YEAR 2023.
---
THERE ARE QUITE A NUMBER OF
THESE PROGRAMS.
THE FIRST MENTION THAT I WANT
TO SAY HERE IS THAT THE
RESEARCH PROMOTED BY THESE
PROGRAMS IS TIED INTO THE
PRINCIPLES THAT I MENTIONED
EARLIER, MULTIPLE POTENTIAL
PATHWAYS TO DEMENTIA IN
POPULATIONS AND INDIVIDUALS
AND YOU CAN SEE THAT
REFLECTED IN THE THEME:
SOCIAL DETERMINANTS,
MODIFIABLE RISK, MECHANISMS
AND PATHOLOGY, MICROBIOME,
CVOID-19 AND DEMENTIA AND SO
ONB.
WORKFORCE DEVELOPING TRAINING
PROGRAMS, FOR DIVERSITY
TRAINING AND CAREER AND FOR A
COMPLETE LISTING PLEASE VISIT
THE NIDS FOCUS ON THE ADRD/AD
PAGE.
AND I WAS A LITTLE BIT MORE
ABOUT THE OPEN ANNOUNCEMENTS.
---
FOR THESE FUNDING
ANNOUNCEMENTS THERE'S A LOT
OF INFORMATION HERE AND I
WON'T GO OVER IT ALL IN
DETAIL. I AM HAPPY TO SHARE
PDF OF THE INITIAL SLIDES
WITH ANYONE AND PLEASE DO
ASK.
I DO WANT TO MENTION THAT THE
ANNOUNCEMENTS ARE LISTED IN
THE ORDER OF RECEIPT DATES.
SO IF YOU DO A SCREENSHOT OR
AS YOU LOOK AT THESE, THE
FIRSTDUE DATES ARE LISTED
FIRST AND THE LATER DUE DATES
ARE LISTED LATER AND ON THE
SLIDES ARE THE DISEASE
MECHANISMS FOAS LISTED IN
ORANGE AND THERE ARE NINE OF
THEM AND FOUR ON TRAINING
LISTED IN BLUE HERE.
---
WE ALSO HAVE INITIATIVES ON
CLINICAL TRIALS, CLINICAL
RESEARCH AND TRANSLATION.
WITH THREE FOUR TRANSLATION
AND FIVE INITIATIVES IN
CLINICAL TRIALS.
---
I ALSO WANT TO SAY AS WE GO
OVER VERY QUICKLY THE SLIDES
THAT SHOW THE INITIATIVES
THAT NO RFA IS NEEDED TO
APPLY.
THE NIDS SPECIAL AD/ADRD
PALIN FOR INVESTIGATOR
INITIATED RESEARCH TO SHARE
WITH THE NATIONAL INSTITUTE
ON AGING AND WE WANT AND WE
HOPE FOR YOUR OWN IDEAS AND
YOUR OWN APPLICATIONS ABOUT
WHAT YOU CONSIDER TO BE THE
MOST IMPORTANT RESEARCH TO
MOVE THE FIELD FORWARD.
AND YOU CAN APPLY UNDER BOTH
THE PARENT RO1 AND THE NIDS R
21.
---
IN SUMMARY, I JUST WANT TO
MENTION THAT I HOPE THAT YOU
HAVE SEEN THE NINDS AD/ADRD
SUMMIT HAS SET NATIONAL
RESEARCH PRIORITIES AND WE
HAD 70 FUNDING INITIATIVES.
THE RESULTS WERE TO ESTABLISH
A NEW MAJOR ADRD PROGRAMS AND
CONSORTIA IN THE NINDS ADRD
HAS GUIDED CONSERVATIVE
RESEARCH AND CONTRIBUTED TO
NEW ADVANCES HOPEFULLY TO THE
DEVELOPMENT OF NEW
INTERVENTIONS.
---
THERE ARE MANY PEOPLE
INVOLVED IN THIS PROGRAM AND
I WANT TO GIVE THANKS FOR THE
TREMENDOUS EFFORTS OF NINDS
AND NIA LEADERSHIP AND STAFF
TODAY I WANT TO CALL OUT THE
NINDS LEADS FOR THE ADRD
SUMMIT SESSIONS LISTED HERE.
FOR AFTER THE, TOM CHEEVER,
FOR HEALTH EQUITY, RICHARD
BENSON, FOR LBD, DEBRA
BABCOCK, FOR MED-OVERALL -TBI
AND -LATE, DOUBLE DUTY, LINDA
MCGAVERN AND MED-CVOID-19,
KEITH WHITAKER.
---
AND WITH THAT I WOULD LIKE TO
WELCOME DR. NATALIA ROST WAS
GOING TO DO THE ADA SUMMIT
2022 REPORT TO THE NINDS
COUNCIL.
THANK YOU.
>> THANK YOU ROD, AND THANK
YOU EVERYONE FOR THE
OPPORTUNITY TO PARTICIPATE.
LOOKING AT THOSE PICTURES
REMINDS ME OF HOW MUCH OF A
GREAT AND WE HAD AT THE
SUMMIT, AND REALLY IT'S AN
HONOR OF MY LIFE. FOR THOSE
OF YOU WHO I HAVE NOT MET MY
NAME IS NATALIA ROST,
SCIENTIFIC CHAIR OF THE 2022
ADRD SUMMIT.
IT WAS A SIGNIFICANT
UNDERTAKING BUT ALSO A GREAT
OPPORTUNITY TO GET TO REALLY
UNDERSTAND THE NUTS AND BOLTS
OF THE PROCESS, AND THE
TRANSPARENCY OF THE PROCESS
BY WHICH THE PRIORITIES IN
AD/ADRD RESEARCH ARE SET. IN
ON BEHALF OF MY ENTIRE TEAM
INCLUDING MY NINDS PARTNER
DOCTOR CORVELL, (PHONETIC),
COMMITTEE CHAIRS AND OTHER
MEMBERS IT IS MY GREAT HONOR
TO PRESENT TO YOU THE ADRD
SUMMIT 2022 REPORT.
---
THE GIST OF THE PRESENTATION
IS THAT THE 22 ADRD SUMMIT
WAS A GREAT SUCCESS AND AS
YOU KNOW IT WAS THE FIRST
VIRTUAL SUMMIT HELD THROUGH
VIDEOCONFERENCING DUE TO THE
LINGERING EFFECTS OF THE
CVOID-19 PANDEMIC BUT DESPITE
THE CHALLENGES WE MANAGED TO
GET THE PROGRAM UP AND READY
FOR PRESENTATION TWO DAYS OF
THE SUMMIT MARCH 22 AND 23.
AND IT WAS A GREAT
ORGANIZATION, STRIVING
TOWARDS GETTING EVERYTHING
READY AND WE WERE EXCITED TO
KNOW THAT THERE WAS A BROAD
STAKEHOLDER ENGAGEMENT ALONG
THE WAY. WE HAD MORE THAN
1500 REGISTERED ATTENDEES AND
DURING THE SUMMIT WE HAD
ANYWHERE BETWEEN 400-700
ATTENDEES PARTICIPATING
DURING MOST SESSIONS. WE HAD
A VERY BROAD ENGAGEMENT
ACROSS THE ACADEMIC CLINICAL
GOVERNMENT, INDUSTRY,
NONPROFIT AND PUBLIC SECTORS
INVOLVED IN THE TWO DAYS OF
PROBLEMS WE HAD8 SCIENTIFIC
SESSIONS WITH 128 PANEL IS
PARTICIPATING AND 44
INDIVIDUAL TALKS AND I AM
PARTICULARLY PROUD TO LET YOU
KNOW THAT WE HAD 210 MINUTES
OF OPEN MIC DISCUSSION SETS
WHICH WERE ALL VERY ROBUSTLY
USED UP.
IT WAS A TREMENDOUS
CONTRIBUTION TO THE CONTENT
OF THE RECOMMENDATIONS AND
REALLY FOSTERING ROBUST
DIALOGUE BETWEEN THE
SCIENTISTS IN PUBLIC AND
OTHER STAKEHOLDERS.
---
WE ALSO-- WHICH IS A NEW AND
I THINK A VERY POSITIVE
DEVELOPMENT-- WE HAD A STRONG
VOICE OF PATIENTS AND
CAREGIVERS IN THE SUMMIT
ABOVE AND BEYOND WHAT HAS
BEEN DONE BEFORE PARTICULARLY
THROUGH THE CONTRIBUTION OF
THE OFFICE OF COMMUNICATIONS
AT NINDS WHICH I AM GRATEFUL
TO FOR CREATING THE VOICES OF
THE ADRD TESTIMONIALS, AND
THE INPUT FROM THE PATIENTS
DURING THE SUMMER WHICH WAS A
GREAT CONVERSATION LEAD THEY
PROVIDED.
I ALSO ENCOURAGE YOU TO
REVIEW THE RECORDINGS AT YOUR
LEISURE, AT THE
VIDEOCAST.NIH.GOV WEBSITE.
WITH COVERED A BROAD ROUND
WITHIN THE ADRD COVERING
TRADITIONAL TOPICS OF HEALTH
EQUITY AND ADRD FROM
DISTEMPORAL SITUATIONS AS
WELL AS OTHER THINGS THAT ROD
OUTLINED.
THE TOPIC INCLUDES THE
PRIVATE SPECIAL TOPICS WITHIN
NIND PLUS TDI AND -- AND TDP
PATHOLOGIES AS WELL AS THE
NEW EMERGENT SESSIONS ON
CVOID-19.
---
I WOULD LIKE TO BEGIN WITH A
BIT OF A GLOBAL VIEW OF WHY
OUR WORK HERE IS SO
IMPORTANT, AND REMIND
EVERYBODY THAT DESPITE THE
RECENT ADVANCES THERE IS
CERTAINLY MUCH MORE TO BE
DONE. I FIND THIS RECENT MAP
GENERATED BY THE GLOBAL
BURDEN OF DISEASE
COLLABORATIVE, AND ESTIMATED
PERCENTAGE CHANGE OF
ESTIMATED DEMENTIA IN THE
WORLD, SHOWING THAT IT IS AN
ONGOING CHALLENGE EVEN SOME
REPORT SHOWING FLATTENING OF
DEMENTIA INCIDENTS WE KNOW
THAT IT IS UNIFORMLY
PROJECTED THAT THE GROWTH IN
PREVALENCE OF DEMENTIA CASES
BETWEEN 2019 AND 2025 IS
EXPECTED AROUND THE WORLD AND
SOME OF THE SMALLEST
ESTIMATES VARY BETWEEN 20-40%
AND THE LARGEST ONES UP TO
2000% FOR THE US PROJECTED TO
BE IN THE 80-100% INCREASE BY
2050, THIS IS VERY
SIGNIFICANT AND DESPITE THE
NEED FOR GLOBAL VIGILANCE AS
CLINICIAN SCIENTISTS MY FOCUS
PERSONALLY-- AND I KNOW THIS
IS THE FOCUS OF THE DEMENTIA
SCIENCE --IS TO REALLY STAY
ON THE NEEDS AND DEVELOPMENT
OF THE INDIVIDUAL PATIENT'S
DISEASE COURSE AND THE
DEMENTIA SCIENCE PARADIGM,
WHERE THE SCIENCE GOES FROM
BEDSIDE TO BENCH AND BACK TO
BEDSIDE WAS ESSENTIAL FOR THE
PREPARATION FOR THE ADRD 2022
SUMMIT AND A SCIENTIFIC SHARE
ON NATURE TO EMPHASIZE THE
DEPERSONALIZATION AND
DIAGNOSTICS INTERVENTION,
DIAGNOSTICS, CARE ARE THE
ULTIMATE GOALS TO MAKE THE
RELEVANT AND AS YOU KNOW, AND
AS DOCTOR CORRIVEAU INDICATED
THIS TASK REMAINS TO BE
COMPLEX. THERE HAS BEEN A
FASCINATED EVOLUTION OF
DEMENTIA AS A CLINICAL
SYNDROME CONCEPT AS YOU HAVE
ALREADY HEARD, ESPECIALLY
OVER THE PAST FEW DECADES.
WITH ACCUMULATING KNOWLEDGE
OF ABOUT UNDERLINING DISEASE
PATHOLOGY AND VARIABILITY OF
CLINICAL FEATURES,
MOLECULAR GENETICS THAT MAKES
THIS SHIFT WHILE THE SIZE
CONTINUES TO EVOLVE PRIOR
GOVERNMENTS LIKE
1PATHOLOGY=1DISEASE IS
RAPIDLY CHANGING AND THERE
ARE MULTIPLE PATHWAYS TO
CLINICAL DEMENTIA SYNDROME.
---
FURTHERMORE WE UNDERSTAND
THAT ON THE WHITE SPECTRUM
BETWEEN BRAIN HEALTH AND ONE
AND IN THE RAIN DISEASE SUCH
AS ADRD AND THE OTHER, THERE
IS LITERALLY A LIFETIME WHERE
EVERY STAGE OF OUR INDIVIDUAL
CHRONOLOGICAL LIFESPAN,
INCLUDING THE INFLUENCES THAT
OCCUR DURING EMBRYOLOGICAL
BRAIN DEVELOPMENT THERE ARE
INNATE AND ENVIRONMENTAL
FACTORS THAT IMPACTED RISK OF
DEVELOPING DEMENTIA AT A
LATER AGE.
AND MOST OF THESE LIFESTAGES
ARE ARBITRARY OBVIOUSLY IN
MANY FACTORS CONTRIBUTE TO
THE INFLUENCE OF THE LIFE
COURSE INCLUDING THOSE THAT
HAVE CUMULATIVE EFFECTS SUCH
AS HEALTH DISPARITIES RELATED
TO HISTORICAL SOCIETAL
STRUCTURES.
---
SO UNSURPRISINGLY TO OVERCOME
THE COMPLEXITIES OF THE ADRD
DISEASE PROCESS OUR SIGNS HAS
TO REMAIN DYNAMIC, ROBUST AND
INFORMED BY PATIENT
EXPERIENCES AND STAYING ON
MISSION TO BRING TOGETHER
EXPERTISE, CROSSCUTTING
SCIENCE AND A PERPETUAL CYCLE
OF INNOVATION, COLLABORATION,
COMMUNICATION AND RESEARCH
AND THAT IS WHAT THE 2022
ADRD SUMMIT WAS ALL ABOUT.
---
BEFORE I SOON ON EVERY
INDIVIDUAL RECOMMENDATION I
WANT TO HIGHLIGHT SOME OF THE
OVERARCHING SESSION SPECIFICS
AND CROSSCUTTING THEMES.
SOME MONETIZATION HIGHLIGHTS
I WANTED TO BRING TO YOUR
ATTENTION OUR HEALTH EQUITY
AND ADRD WAS A CENTRAL
DISCUSSION.
AND THE CONCEPT OF INCLUSION
SCIENCE, WITH INCLUSION
BROADLY INVOLVING STUDY
PARTICIPANTS, WORKFORCE THAT
PARTICIPATE IN RESEARCH,
COMMUNITIES AND ALSO SOCIETY
HAS BEEN FRONT AND CENTER.
WITH FTD THEY HAVE BEEN
QUITE A FEW CRITICAL ADVANCES
IN BIOMARKERS DEVELOPMENTS
AS WELL AS TRANSLATION TO
CLINICAL TRIALS AND ATTEMPTS
TO ADDRESS THE BROAD SPECTRUM
OF THE DISEASE HAS BEEN ONE
OF THE LEADING ONCE ACROSS
THE BOARD AND WITHIN VCID,
BIOMARKERS TO UNRAVEL COMPLEX
PHENOTYPES ARE TAKING
PRIORITIES AND CENTRAL
STATES AS WELL AS THE
DEVELOPMENT OF NOVEL
DEPARTMENTAL MODELS AND
TRANSLATION OF THE BASIC
RESEARCH FINDINGS INTO
CLINICAL SCIENCE.
---
IN LBD THE CENTRAL EFFORTS
ARE AROUND CLINICAL
CHARACTERIZATION AND
INTERVENTION AS WELL AS
PATHOGENESIS AND MECHANISMS
OF TOXICITY. AND MED AGAIN
BEING ONE OF THE BROADEST
CATEGORIES, MANAGED TO STAY
QUIET FOCUS ON DETECTION
AND DIAGNOSIS, INTERVENTIONS
AND TREATMENTS, AS WELL AS
DATA HARMONIZATION AND
DEMENTIA CAPABLE WORKFORCE.
AND WITHIN THE MED SPECIAL
SUBTOPICS, POST-TBI AD/ADRD,
COLLABORATION, HARMONIZATION
AND PHENOTYPE
CHARACTERIZATION OF SOME OF
THE CENTRAL PRIORITIES.
TDP-43 IN COMMON AND LATE
ONSET DEMENTIAS IS WORKING ON
DEFINITION, 2 LANDMARKS AND
DISEASE MODELS AND THE022
EMERGING SCIENCE PROBABLY
REVIEWED THE IMPACT OF
CVOID-19 AND AD/ADRD RISK AND
OUTCOMES.
---
SO DESPITE EACH SESSION
HAVING THEIR OWN PRIORITIES
AND OWN CENTRAL THEMES, THE
CROSSCUTTING THEME IS WHAT
PUT THE OVERARCHING GOAL OF
THE ADRD SUMMIT IN 2022
TOGETHER.
SOME OF THE CROSSCUTTING
THEMES I WANTED TO HIDE IT I
REALLY THE HEALTH EQUITY IN
AD/ADRD AS A MAJOR IMPERATIVE
AND THE CONTINUING UNMET NEED
IN RESEARCH.
THE NEED FOR RESEARCH AND
IMPLEMENTATION OF PRAGMATIC
APPROACHES AND SOLUTIONS IN
AD/ADRD, INCLUDING PRAGMATIC
CLINICAL TRIAL DESIGNS.
THE URGENT NEED FOR PRECISE
BIOMARKERS ACROSS EVERY
DISEASE CATEGORY WITHIN
AD/ADRD TO IDENTIFY
UNDERLINING DISEASE PROCESSES.
HELPING INDIVIDUALS OR THOSE
WHO ARE DIAGNOSED WITH
EITHER ADRD OR QUADROMAL
(CORRECTION) PRODROMAL
SYNDROMES.
INNOVATIVE APPROACHES TO
PERSONAL PREVENTION.
IN CLINICAL RESEARCH,
PREVENTION, PRESYMPTOMATIC
VIGILANCE, AND IN BASIC
RESEARCH NEEDS INCLUDE NOVEL
STRATEGIES AND TOOLS IN
SEEKING SYNERGIES MAXIMIZING
THE DIVERGENCE INDICES
PATHWAYS IN ORDER TO PROCEED
TO FUTURE ADVANCES.
---
SO I WILL NOW SPEND A FEW
MINUTES HIGHLIGHTING TOP
PRIORITIES AND
RECOMMENDATIONS BY EACH
INDIVIDUAL SCIENTIFIC WORKING
GROUP AND I WILL START WITH
THE HEALTH EQUITY IN ADRD AND
PROCEEDING THE ORDER IN WHICH
THE RECOMMENDATIONS WERE
PRESENTED.
---
AS YOU KNOW AND I HAVE
ALREADY MENTIONED TO YOU THE
HEALTH EQUITY WORKING GROUP
HAS PRESENTED A CLEAR VISION
FOR ACHIEVING AD/ADRD HEALTH
EQUITY THROUGH RESEARCH WITH
A CENTRAL FOCUS ON DIVERSITY
IN THE WORKFORCE AND
INCLUSION SCIENCE FRAMEWORK.
THE TOP PRIORITIES PRESENTED
BY THE WORKGROUP LED BY
DOCTOR GONZALEZ AND
ZISSIMOPOULOS CENTERED ON
ADVANCING EQUITY AND
INCLUSION OF SCIENCE TO
IMPROVE REPRESENTATION
SAMPLING AND INCREASE
TRAINING AND SUPPORT AND
CAPACITY OF AN AD/ADRD
SCIENTIFIC WORKFORCE OF
PERSONS HISTORICALLY
UNDERREPRESENTED IN
BIOMEDICAL, BEHAVIORAL AND
SOCIAL SCIENCES.
---
THE FRONTOTEMPORAL DEMENTIA
AS A FIELD HAS EXPERIENCED
RAPID ADVANCEMENT OVER THE
PAST FEW YEARS. THIS
INVESTMENT HAS BEEN LARGELY
FUELED BY THE LARGE-SCALE
NATIONAL AND INTERNATIONAL
COLLABORATION SUCH AS OFTD
AND THE FTD PREVENTION
INITIATIVE LEADING TO
BIOMARKER DEVELOPMENT AND
SOME OF THE CLINICAL TRIALS
IN FTD.
AND THE WORKING GROUP LED BY
DOCTORS BOXER AND KARCH
FOCUS ON UNDERSTANDING FTD
EPIDEMIOLOGY AND GENETICS IN
DIVERSE POPULATIONS INCLUDING
HOW SOCIOECONOMIC AND
ETHNOCULTURAL STATUS AFFECT
DISEASE RISK AND
MANIFESTATIONS AS WELL AS IN
ADVANCING UNDERSTANDING OF
FTD AND IDENTIFY THERAPEUTIC
TARGETS.
---
VCID IS AN X WORKING GROUP I
WOULD LIKE TO HIGHLIGHT. VCID
IS ONE OF THE MOST RECOGNIZED
AND PREVENTIVE FORMS OF BRAIN
DISEASE ARE MORE OFTEN THAN
NOT IN THE POPULATION THAT
ARE CONSIDERED TO BE
"DISEASE-FREE," ONTO THE
CRITICAL BURDEN OF DISEASE
CIRCULATION LEADS A
RELENTLESS COGNITIVE DECLINE.
DESPITE THE HETEROGENEITY AND
PATHOLOGICAL CHARACTERISTICS,
VCID IS ALSO THE MOST
PREVENTABLE FORM OF DEMENTIA
AT LEAST AT THIS TIME AND
AGE.
AND THE DEMENTIA CAN BE
ATTACKED VIA MULTIPLE
MECHANISMS THAT OVERALL HAVE
A POSITIVE MULTISYSTEM EFFECT
INCLUDING THE CARDIOVASCULAR
AND BRAIN HEALTH. THE VCID
WORKING GROUP COCHAIRED BY
DOCTORS WOLCOCK AND PETERSEN
FOCUS ON ESTABLISHING AND
REFINING EXPERIMENTAL MODELS
AND TECHNOLOGIES TO IDENTIFY
DISEASE RELEVANT MECHANISMS
UNDERLYING VCID.
AS WELL AS DEVELOPING AND
VALIDATE MARKERS
IN DIVERSE POPULATIONS USING
COGNITIVE AND OTHER
BIOMARKERS AS WELL AS
BIOMARKERS OF KEY VASCULAR
PROCESSES INCLUDING IN THE
MOST COMMON SCENARIO WHERE
VCID IS ACCOMPANIED BY AD IN
HUMAN STUDIES.
---
THE NEXT WORKING BUT WOULD
LIKE TO HIGHLIGHT IS THE LEWY
BODY DEMENTIAL, ANOTHER
HIGHLY DISABLING ADRD
CATEGORY OF DEMENTIA BUT ALSO
THE FIELD THAT TOOK ON THE
COMPLEXITY THAT UNDERLINES
THE DISEASE LARGELY DOMINATED
BY THE-- PATHOLOGIST AND THE
WORKING GROUP LED BY DOCTORS
LEVERENZ AND KANTARCI SHARE
THE RECOMMENDATIONS INCLUDING
THE PARITY TO PREPARE AND
INITIATE CLINICAL TRIALS THAT
AIM TO ALLEVIATE OR SLOW THE
COURSE OF LBD SYMPTOMS AND
DELAY OR PREVENT THE ONSET OF
DISEASE.
ONE OF THE PRIORITIES THAT
ALIGNS VERY CLOSELY WITH THE
PUBLIC STAKEHOLDERS' INPUT.
---
ANOTHER TOP PRIORITY IN THE
WORKGROUP IS A RECOMMENDATION
TO DELINEATE GENETIC LOCI
AND THEIR FUNCTIONS
CONTRIBUTING TO THE ONSET AND
PROGRESSION OF LBDS USING
GENETIC, TRANSCRIPTOMIC
ANALYSIS.
---
AND FINALLY MULTIPLE OR MIXED
ETIOLOGY DEMENTIAS, MED
ITSELF REMAINS PERHAPS THE
MOST COMPLEX IF NOT THE
UNIFYING CATEGORY. GIVEN
THAT MOST INDIVIDUALS
DIAGNOSED WITH DEMENTIA ARE
KNOWN TO HAVE MIXED
PATHOLOGIES. BUT THE MED
WORKING GROUP REALLY
MAINTAINED WITH A MORE
COMPREHENSIVE APPROACH IS TO
THE RECOMMENDATIONS FOCUSING
ON DISEASE DETECTION AND
DIAGNOSIS AS WELL AS
INTERVENTION AND TREATMENT
FUELED BY BASIC SCIENCE AND
SUPPORTED BY DATA
HARMONIZATION AND SUSTAINING
THE DEMENTIA CAPABLE
WORKFORCE, A VERY IMPORTANT
FOCUS OF THE WORKBOOK.
---
IN THIS CONTEXT THE WORKGROUP
LED BY DOCTOR POSSIN
WORK TO EVALUATE PRAGMATIC
APPROACHES TO OBJECTIVELY
DETECT COGNITIVE IMPAIRMENT
AND LINK TO QUALITY CARE WHEN
THE PATIENT, CARE PARTNER,
COMMISSION REPORTS COGNITIVE,
BEHAVIORAL OR FUNCTIONAL
CHANGES IN THE SECOND
PRIORITY IS TO CONDUCT
CLINICAL STUDIES ON APPROVED
A PROMISING INTERVENTIONS AND
TREATMENT TO MITIGATE RISK
FOR COGNITIVE THE CLIENT.
AND WITHIN THE FRAMEWORK OF
MED THERE ARE SPECIAL TOPICS
AS YOU HEARD.
TWO SPECIAL TOPICS HAVE BEEN
ADOPTED TOWARD PRIOR ADRD
SUMMIT OF 2019, WHICH ARE
CURRENTLY THE TWO INDIVIDUAL
SPECIAL TOPICS THAT HAVE BEEN
PRESENTED THERE, WITH
INDIVIDUAL TOP PRIORITIES AND
RECOMMENDATIONS.
THE POST TRAUMATIC INJURY
ADRD OR POST-TBI ADRD GROUP
CHAIRED BY DOCTOR O'CONNOR,
PROMOTED COLLABORATION AMONG
TBI AND DEMENTIA RESEARCH IS
TO WORKING GROUPS,
RETROSPECTIVE AND PROSPECTIVE
DATA AND MEASUREMENT
HARMONIZATION AND
INTERDISCIPLINARY RESEARCH,
WITH PATHOLOGY MAKING GREAT
STRIDES IN SHOWING
SIGNIFICANT ADVANCES IN
SCIENCE IN THE PAST SEVERAL
YEARS.
---
THE SECOND SPECIAL TOPIC, TDP
43 IN COMMON LATE-CONCEPT
DEMENTIAS WAS CHAIRED BY
DOCTOR SCHNEIDER WHO
PRESENTED THEIR PRIORITIES IN
DEFINING LATE, WHICH IS A
CLEVER ACRONYM FOR THE
LIMBIC PREDOMINANT
AGE-RELATED TDP-43
ENCEPHALOPATHY,
AND DEFINING THE PATHOLOGIC,
CLINICAL, GENETIC AND
MOLECULAR CLASSIFICATION AND
DIAGNOSTIC BOUNDARIES.
AND FINALLY THE EMERGING
SIGNS WITHIN THE SPECIAL
TOPICS WAS THE MUCH
ANTICIPATED PRESENTATION ON
THE IMPACT OF CVOID-19 ON THE
AD/ADRD RISK AND OUTCOMES.
OBVIOUSLY OUR LIVES HAVE BEEN
TREMENDOUSLY DOMINATED BY THE
TOPIC OF CVOID-19 OVER THE
PAST SEVERAL YEARS AND THIS
TOPIC WAS WORTHY OF A SPECIAL
AND TRULY EMERGING SCIENCE
STATUS IN 2022.
---
AS YOU KNOW THERE ARE NEARLY
94 MILLION SARS-COV-2
INFECTION SURVIVORS IN THE
UNITED STATES ALONE. WHAT WE
AS CLINICIANS LEARNED FROM
THE BEGINNING OF BATTLE WITH
HIS INFECTION IS THAT
NEUROLOGICAL,
NEUROPSYCHIATRIC AND
COGNITIVE COMPLAINTS ARE
COMMON AMONG SURVIVORS AND WE
HAVE LEARNED THAT THE
SARS-COV-2 INFECTION HAS
LONG-TERM IMPACT ON THE BRAIN
INCLUDING THE POTENTIAL
DOWNSTREAM EFFECT ON
NEURODEGENERATIVE AND
VASCULAR INJURY PATHWAYS AND
WHILE THE OVERALL IMPACT OF
AD/ADRD RISK AND OUTCOMES
REMAINS UNKNOWN AT THIS POINT
THE POTENTIAL DETRIMENTAL
EFFECTS OF AD/ADRD MAY
INCLUDE INCREASED INCIDENCE
OF COGNITIVE IMPAIRMENT AND
DEMENTIA CASES IN THE US AND
WORLDWIDE AS TIME GOES BY.
ACCELERATED COURSE OF
COGNITIVE DECLINE AMONG THOSE
ALREADY DIAGNOSED WITH
AD/ADRD, A NEW CATEGORY.
ACCELERATED COURSE OF
NEURODEGENERATION AND BRING
VASCULAR PATHOLOGY AMONG
ALREADY DIAGNOSED OR
ASYMPTOMATIC POPULATIONS.
A SHIFT IN THE POPULATION
TIMELINE OF COGNITIVE
IMPAIRMENT/DEMENTIA CLINICAL
ANSWER TO HER YOUNGER AGES
AND THEREFORE INCREASING
INCIDENCE OF EARLY ONSET AS
WELL AS INCREASE MORTALITY
AMONG INDIVIDUALS WITH
ADVANCED DEMENTIA.
---
AT THIS TIME THE DATA STILL
SCARCE AND KNOWLEDGE IS
RAPIDLY EVOLVING.
AS SEEN AND RECENTLY
PUBLISHED A NUMBER OF
STUDIES, ONE OF THEM ON THE
LEFT, THEY EXAMINE 400
PATIENTS WERE TESTED POSITIVE
FOR CVOID-19 AND SHOWED UP
REASONABLE CHANGES IN THE
BRAIN STRUCTURE CORRESPONDENT
DECLINE AND NOTABLY THE
CARETAKERS AS SHOWN IN A
RECENT INTERNATIONAL
COLLABORATION PUBLISHED IN
2022 IN THE STUDY ON THE
RIGHT.
---
SO WHILE WE HAVE A LOT OF
QUESTIONS TO ANSWER IN THE
FUTURE RELATED TO THE SPECIAL
TOPIC, AND THE WAY IT RELATES
TO THE REST OF THE POST-
(INDISCERNIBLE) OF COVID AS
PRESENTED EARLIER OR LONG
COVID SYNDROME THAT IS KNOWN
TO HAVE COGNITIVE FEATURES IS
THE 2022 SCIENTIFIC SHARE
HAD THE PRIVILEGE TO FORM A
SPECIAL TOPIC WORKING GROUP
IN COLLABORATION WITH DOCTOR
SUDHA SESHADRI, AND PRESENTED
OUR INITIAL RECOMMENDATIONS
PUTTING THE RECOMMENDATION TO
ESTABLISH RESEARCH
INFRASTRUCTURE ENABLING
CLINICAL, EPIDEMIOLOGICAL AND
BASIC RESEARCH STUDIES OF
CVOID-19 IMPACT ON RISK AND
OUTCOMES PRIORITIZING
DISPROPORTIONATELY AFFECTED
POPULATIONS AND CLINICAL
TRIALS READINESS, THIS IS ONE
OF THE PRIORITIES THAT IS
CLOSELY DEVELOPED WITH OTHER
WORKING GROUPS PARTICULARLY
EFFECTIVE IN OUR SUMMIT
PREPARATION.
---
OTHER RECOMMENDATIONS FOR THE
SPECIAL TOPIC INCLUDED
CHARACTERIZATION OF THE
CLINICAL PHENOTYPE AND
DEVELOPING DIAGNOSTIC
CRITERIA FOR NEUROCOGNITIVE
IMPAIRMENT AND DEMENTIA
ASSOCIATED WITH COVID.
EXPLORING INTERACTIONS OF
SOCIAL, STRUCTURAL, AND
SYSTEMIC INEQUALITIES,
COMORBIDITIES AND SOCIAL AND
MEDICAL INTERVENTIONS WITH
RISK AND YOUR COGNITIVE
SEQUELA OF CVOID-19.
AND ADVANCED UNDERSTANDING
THE BASIC MECHANISMS OF
ALIGNING NEUROCOGNITIVE
IMPAIRMENT AND DEMENTIA DUE
TO CVOID-19, AND THE
FOUNDATION FOR EARLY
INTERVENTION TRIAL SO I
ENCOURAGE YOU TO STAY TUNED
TO THIS TOPIC WE ARE EXCITED
TO INCLUDE IT WITHIN THE 2022
ADRD SUMMIT CONTEXT.
---
SO THE SUMMIT WAS A TERRIFIC
OPPORTUNITY TO LEARN MORE
ABOUT SCIENTIFIC ADVANCES, TO
DISCUSS AND DEBATE THE
PERCEIVED GAPS AND IDENTIFY
TOP PRIORITIES AND THE WAYS
TO MOVE TOWARDS BOTH THE
NATIONAL PLAN GOALS AS WELL
AS THE BENEFIT OF OUR
PATIENTS, THEIR FAMILIES AND
SOCIETY AT LARGE. I WANTED TO
THANK MY FANTASTIC TEAM IN
PLANNING AND IN THE
EXECUTION OF SOME
SPECIFICALLY THE ADRD CORE
GROUP AND A DEDICATED SUPPORT
STAFF, DR. WHITAKER AND DR.
MCCARTNEY.
A BIG GREAT THANKS.
I WOULD LIKE TO RESPECTFULLY
UNDERSCORE THE OLD
RECOMMENDATIONS IN THE REPORT
REPRESENT CRITICAL NATIONAL
RESEARCH PATHWAYS THAT ARE
TOP SCIENTIFIC PRIORITIES
TOWARD THE GOAL OF ACHIEVING
EFFECTIVE PREVENTION AND
TREATMENT OF AD AND AD/ADRD.
AND WITH THE INPUT OF THE
MULTIPLE STAKEHOLDERS AND THE
PUBLIC, ON BEHALF OF THE
COMMITTEE COCHAIRS, MEMBERS
T WE RESPECTFULLY SUBMITHE
ADRD SUMMIT 2022 REPORT.
THANK YOU AND I WILL BE HAPPY
TO ENTERTAIN ANY QUESTIONS.
---
>> THANK YOU THAT WAS GREAT.
I ACTUALLY FORGOT TO MENTION
AT THE BEGINNING AS YOU
POINTED OUT THAT THIS REPORT
WHICH WAS IN THE ECB, THE
COUNCIL ASKED TO FORMALLY
ACCEPT THIS REPORT WHICH DOES
NOT MEAN YOU AGREE WITH EVERY
SINGLE LINE OF IT BUT THAT
YOU BASICALLY ACCEPT ITS
RECOMMENDATIONS. THANKS A LOT
THAT WAS A TREMENDOUS SUMMARY.
I THINK YOU CAN TAKE DOWN THE
SLIDE.
---
ARE THERE QUESTIONS?
>> I JUST WANT TO THANK THEM
FOR THE TREMENDOUS EFFORT PUT
INTO THIS. IS A BEAUTIFUL,
BEAUTIFUL SUMMARY, THANK YOU.
>> DR. FINKELSTEIN: IT'S A
HUGE UNDERTAKING.
TIM?
>> MY QUESTION WAS TO ROD.
SINCE YOU BOTHERED TO SHOW IS
THE EXCITING DATA.
WHAT FRACTION OF PATIENTS
CARRIED THAT TRANSLOCATION?
>> DR. CORRIVEAU: THAT
PARTICULAR ONE TIM IS NOT
GOING TO BE A VERY LARGE
NUMBER. I THINK THE OVERALL
POINT THOUGH IS A PRINCIPLE
OF THE POSSIBILITY OF THIS
TYPE OF INTERVENTION AND
PURSUING IT.
>> OKAY.
>> DR. CORRIVEAU: IT WILL NOT
BE A LARGE NUMBER.
>> THANK YOU.
>> DR. FINKELSTEIN: BOB?
>> >> DR. BROWN:
A COUPLE OF THINGS, FIRST OF
ALL THANK YOU NATALIA FOR THE
FANTASTIC ORAL REPORT AND
ALSO FOR THE WRITTEN DOCUMENT
WHICH IS STATE OF THE ART AND
FANTASTIC.
I WANT TO COMMENT BRIEFLY ON
WHAT WAS JUST BROUGHT UP, THE
ISSUE OF SUPPRESSION OF
DOMINANT REACTING
NEURODEGENERATIVE GENES.
IT HAS NOW BEEN DEMONSTRATED
OVER THE LAST TWO OR THREE
YEARS THAT IN HUMAN PATIENTS
IS POSSIBLY SUPPRESSED NOT
ONLY FOS FOR ALSO C9 OR C72
WHICH RELATE TO BOTH ALS AND
FTD.
THE ISSUE IS THAT MUCH AS WAS
THE CASE WITH NEIL
SCHNEIDER'S CASE, THIS DOES
NOT MAKE PEOPLE BETTER, IT
PROBABLY SLOWS THE DISEASE A
BIT.
THE ISSUE IS THAT THIS
EMPHASIZES TWO THINGS. FIRST
OF ALL AS NATALIA ALLUDED, TO
UNDERSTANDING DOWNSTREAM
EVENTS AND NOTABLE POINTS
WHICH ARE PARTICULARLY GOOD
ACHILLES HEELS FOR THERAPY
INDEPENDENTLY OF SHUTTING OFF
THE TRIGGER WHICH HAS TO BE
DONE WHEN THERE IS A TRIGGER.
I GET THAT.
---
BUT ALSO ANOTHER POINT THAT
MIGHT BE WORTH EMPHASIZING
MORE-- AND SHE DID ALLUDE TO
THIS-- THE CRITICAL ASPECT IS
IDENTIFYING EARLY MARGINALLY
OR MINIMALLY SYMPTOMATIC
PATIENTS WITH THESE LESIONS
WHERE WE CAN INTERVENE BEFORE
THE HOUSE STARTS TO BURN UP
AND FIRE.
AND SO WE NEED TO RECOGNIZE
THE POINT BETWEEN
PRESYMPTOMATIC AND
SYMPTOMATIC AND LOOKING FOR
THOSE BIOMARKERS TO IDENTIFY
THE CRITICAL POINT IS SUPER
IMPORTANT AND I COMMEND YOU
FOR POINTING THAT OUT.
>> THANK YOU SO MUCH BOB.
ALSO WANTED TO SAY THAT THIS
ECHOES THE SENTIMENT
TREMENDOUSLY WITHIN THE
PATIENT AND CAREGIVER INPUT
IN EVERYTHING THAT WE
DISCUSSED. THIS PRE-PODROMAL
VIGILANCE FOR THE DISEASE IS
SOMETHING SCREAMING AND DOING
SOMETHING VERY EARLY IN THE
DISEASE STAGES,
IT'S SOMETHING THAT OBVIOUSLY
PATIENTS ARE VERY MUCH
INVESTED IN.
>>
>> DR. FINKELSTEIN: OKAY.
ANY OTHER COMMENTS ABOUT
EITHER ROD'S TALK OR THE
SUMMIT REPORT?
---
WELL THANKS TO BOTH OF YOU.
WE DO NEED A MOTION TO, FOR
EXAMPLE, APPROVE THE REPORT
FROM THE ADRD SUMMIT.
>> SO MOVED.
>> DR. FINKELSTEIN: I SEE A
MOTION AND MANY SECONDS. WHY
DON'T YOU VOTE FOR THAT
MOTION. THANKS AGAIN NATALIA
FOR COMING. THAT WAS REALLY
GREAT, AND ROD FOR HIS USUAL,
LUCID PRESENTATIONS.
>> THANK YOU.
>> DR. FINKELSTEIN: OKAY SO
WE WILL SHIFT GEARS HERE.
AND LET ME SEE.
I GUESS THE LAST THING IS
CONCEPT CLEARANCE. THERE IS
NO BREAK IN THE SCHEDULE NOW
SO WE WILL PLOW RIGHT AHEAD.
SO WE ARE COMING DOWN THE
HOME STRETCH HERE.
BASICALLY AS YOU KNOW, BEFORE
WE LAUNCH ANY NEW INITIATIVE
WE ASK FOR CONCEPT CLEARANCE
FROM COUNCIL WE HAVE ALWAYS
DONE THIS BUT NOW-- DO YOU
HAVE A QUESTION GINA?
>> NOT A QUESTION BUT AN
ANNOUNCEMENT. I WANTED TO SAY
THANK YOU TO NINDS BECAUSE
TOMORROW IS THE DEADLINE FOR
THE VERY FIRST INITIATIVE FOR
THOSE INVESTIGATORS AT RISK,
OR FOR SOME INVESTIGATORS I
WANT TO THANK NINDS FOR THE
TREMENDOUS EFFORT THAT WENT
INTO THAT TOGETHER AND
HOPEFULLY FINGERS CROSSED WE
WILL GET SOME GREAT
APPLICATIONS.
>> DR. FINKELSTEIN: YEAH THIS
IS REALLY AN EFFORT BY
MICHELE JONES LINDON
(PHONETIC) AND HER OPEN
OFFICE, INCREDIBLY IMPORTANT
AND OTHER INSTITUTES WHICH
SIGN ONTO THAT WHICH IS GREAT
AND MY IMPRESSION IS WE ARE
GETTING A PRETTY HEALTHY
RESPONSE BUT I THINK MICHELE
WILL BE TALKING ABOUT THAT IN
A FUTURE COUNCIL MEETING.
THIS IS THE LATEST THING THAT
WE ARE TRYING AND IT SEEMS
THAT IT HAS THE POTENTIAL TO
BE SUCCESSFUL.
---
SO CONCEPT CLEARANCE IS
ACTUALLY MANDATED BY NIH THAT
WE ASKED THE COUNCIL, EVEN
THOUGH WE ALWAYS USED TO DO
IT. A LOT OF THE INITIATIVES
THAT COME THROUGH ARE
ACTUALLY BE ISSUES OF
PREVIOUS INITIATIVES; WE
DON'T USUALLY DISCUSS EACH OF
THESE. SOMETIMES THERE'S A
WHOLE BUNCH OF THEM. BUT
THERE ARE THREE CONCEPTS WE
WOULD LIKE TO PRESENT. IN
FACT THE FIRST ONE IS EVERY
ISSUE OF AN EXISTING PROGRAM
FROM MICHELE JONES LONDON'S
OFFICE. SO WHY DON'T I
INTRODUCE MARGARET MATTHEWS
FROM MICHELLE'S OFFICE WHO IS
GOING TO TALK ABOUT THIS
FIRST INITIATIVE.
ARE YOU RUNNING YOUR SLIDES?
>> YES I WILL BE RUNNING MY
SLIDES.
>> DR. FINKELSTEIN: WELCOME.
>> THANK YOU.
-- --
IS EVERYONE ABLE TO SEE MY
SLIDES IN FULL-SCREEN MODE?
>> DR. FINKELSTEIN:
ABSOLUTELY.
>> WONDERFUL GOOD AFTERNOON
EVERYONE OR I SHOULD SAY GOOD
EVENING AUDIENCES YOU ARE IN
EUROPE AND I'M HAPPY TO TALK
WITH YOU ABOUT THE NIH
NEUROSCIENCE DEVELOPMENT FOR
ADVANCING THE CAREERS OF A
DIVERSE RESEARCH WORKFORCE
WHICH WE SHORTENED AND CALL
THE DIVERSITY >> R 25.
ABOUT TWO YEARS AGO WE
PARTNERED WITH NIMH WITH
JAMES CHURCHILL, TO BROADEN
THE NEUROSCIENCE THAT WE CAN
SUPPORT OF THIS INITIATIVE.
---
FOR THOSE OF YOU WHO MAY OR
MAY NOT KNOW NINDS HAS
EXTENSIVE PATHWAY TRAINING
PROGRAMS THAT SPAN MANY
DIFFERENT CAREER STAGES AND
TRANSITION POINTS, FROM HIGH
SCHOOL ALL THE WAY UP TO
JUNIOR FACULTY. SOME OF THESE
25 MECHANISMS INCLUDE -- THE
PROGRAM SUPPORT UNDERGRADUATE
RESEARCH IN THE SUMMER R 25
PROGRAM SUPPORTS ALSO HUSTLE
STUDENTS AND HIGH SCHOOL
TEACHERS ON THEIR SUMMER
BREAK AND WE RECENTLY
LAUNCHED THANKS TO THE INPUT
FROM THIS GROUP THE DOCTORAL
READINESS PROGRAMS WHICH
TARGETS POSTBACCALAUREATE
STUDENTS IN CONJUNCTION WITH
OUR CURRENTLY FUNDED T 32
PROBLEMS IN THIS NIH
NEUROSCIENCE DIVERSITY R 25
PROGRAMS WHICH SUPPORTS
PRE-DOCTORS UP TO EARLY
CAREER FACULTY AND RECENTLY
WE WERE ABLE TO PUBLISH A
NOTICE OF INFORMATION TO
ENCOURAGE GRANT APPLICATIONS
AND FOCUS ON GETTING MORE
UNDERREPRESENTED RESEARCHERS
IN THE TRANSLATIONAL RESEARCH
SPACE.
---
SO, ONE OF THE REALLY
IMPORTANT HALLMARKS OF THIS
PARTICULAR DIVERSITY R 25
THAT TARGETED PRE-DOCTORAL TO
JUNIOR FACULTY STAGES THAT WE
HAVE THE STUDENTS THAT ARE
ALREADY COMMITTED TO PURSUING
DOCTORAL STUDIES IN
NEUROSCIENCE AND IT IS
IMPORTANT TO HAVE ADDITIONAL
PROFESSIONAL DEVELOPMENT
OPPORTUNITIES FOR THEM TO GET
FURTHER MENTORSHIP OUTSIDE OF
JUST THEIR HOME INSTITUTIONS
OR THEIR PI THAT THEY ARE
WORKING WITH.
BUT IT ALSO CREATES NETWORKS
AND COHORTS AND IT ALLOWS THE
STUDENTS TO BUILD COMMUNITY
WITH OTHER NEUROSCIENTISTS
AND EXPAND THEIR EXPERIENCE.
AND SO THESE ARE JUST A FEW
OF THE PROGRAMS THAT I WOULD
LIKE TO HIGHLIGHT, MANY OF
WHICH ARE LONG-STANDING. A
FEW OF YOU ON THIS COUNCIL
ARE VERY INTIMATELY CONNECTED
WITH AT LEAST ONE OF THESE
PROGRAMS. BOTH MICHELE JONES
LONDON AND I ARE IN FACT
PRODUCTS ARE ONE OF THESE
PROGRAMS AND THEY HAPPEN TO
BE INCREDIBLY PIVOTAL IN HOW
WE HAVE BEEN ABLE TO PROGRESS
IN OUR CAREERS. AND IF ANY OF
YOU ARE ON TWITTER YOU CAN
SEE HOW MUCH OF THESE
COMMUNITIES, THESE MENTORING
OPPORTUNITIES MEAN TO THE
STUDENTS WHO ARE INVOLVED IN
THEM. BUT NOT ONLY IS
PROVIDING AN OPPORTUNITY FOR
INDIVIDUALS.
IT ALSO IS AN OPPORTUNITY TO
CREATE INSTITUTIONAL CHANGE
AND REQUIRE INSTITUTIONAL OR
ORGANIZATIONAL COMMITMENTS TO
INCREASING THE WORKFORCE
DIVERSITY AS WELL AS
INCLUSION.
---
AND SO ONE OF THE GAP AREAS
THAT ARE BEING ADDRESSED
THROUGH THIS PARTICULAR
MECHANISM IS PROVIDING
PROFESSIONAL OPPORTUNITIES
AND NETWORKING OPPORTUNITIES
FOR UNDERREPRESENTED
NEUROSCIENTISTS AND ALSO
ENHANCE INSTITUTIONAL
COMMITMENT TO WORKFORCE
DIVERSITY AND INCLUSION.
FOR THIS PARTICULAR
MECHANISM, THE FOCUS IS ON
GRADUATE STUDENTS,
POSTDOCTORAL RESEARCHERS, AND
JUNIOR FACULTY. SOME OF THESE
PROGRAMS THAT HAVE ALREADY
BEEN FUNDED, AS SHOWN ON THE
PREVIOUS SLIDE, ACTUALLY DO
LOOK AT MULTIPLE CAREER
STAGES AND HELP WITH SOME OF
THESE TRANSITION POINTS.
FOR EXAMPLE THE SOCIETY FOR
NEUROSCIENCE SCHOLARS PROGRAM
ALLOWS BOTH PRE-DOCTORAL
STUDENTS AS WELL AS
POSTDOCTORAL RESEARCHERS TO
BE INVOLVED. THE BRAINS
PROGRAM RUN BY THE UNIVERSITY
OF WASHINGTON HAS JUNIOR
FACULTY BUT REALLY THINKING
ABOUT POSITIONING THEM WELL
TO BE ABLE TO GET THEIR FIRST
R 01S AND ESTABLISH THEIR
LABS. SO WE REALLY THINK
STRATEGICALLY ABOUT THE TYPES
OF PROGRAMS THAT WE ARE
FUNDING THAT ALLOW FOR SOME
OF THESE TRANSITION POINTS OR
BRINGING TOGETHER MULTIPLE
CAREER STAGES TO SERVE AS
EXPERIMENTAL OR HELPING ONE
STATE MOVE TO THE NEXT.
---
THE TYPICAL BUDGET FOR THIS
PROGRAM IS UP TO $250,000 IN
DIRECT COSTS PER YEAR PER
PROGRAM.
AND WE DO EXPECT THAT EACH
PROGRAM, OR EACH APPLICATION
THAT COMES IN, THAT THESE
PROGRAMS ARE FOCUSED ON
OUTLINING BOTH SHORT-TERM AND
LONG-TERM OUTCOMES, AND BEING
ABLE TO EVALUATE SOME
SUCCESSES WHETHER THAT IS THE
TYPE OF PRESENTATIONS, OTHER
OPPORTUNITIES FOR NETWORKING,
PUBLICATIONS, OPPORTUNITIES
FOR GETTING MORE GRANTS AND
THE ABILITY TO TRANSITION TO
THE NEXT CAREER STAGE. SOME
OF THESE PROGRAMS HAVE
PUBLISHED ON THOSE OUTCOMES
AND I AM HAPPY TO SHARE THE
CITATIONS IN THE CHAT ONCE
THIS IS OVER.
---
THAT IS ALL I HAVE.
I AM HAPPY TO TAKE ANY
QUESTIONS.
>> DR. FINKELSTEIN:
YOU CAN TAKE DOWN THE SLIDE.
ANY QUESTIONS OR COMMENTS
ABOUT THIS ONE?
-- --
GINA?
>> I WANT TO SAY THAT THIS IS
AN AWESOME EFFORT AND IT'S
DONE SO MUCH.
THERE ARE HUNDREDS OF PEOPLE
WHO ARE ALUMNI OF THIS WHO
ARE BENEFITING FROM THESE
PROGRAMS IN THE PAST, AND IN
FACT I AM CURRENTLY ON A ZOOM
CALL WITH THE INCOMING NSP
PROGRAM FELLOWS AND I WANT TO
SAY THERE IS SO MUCH
ENTHUSIASM. IT IS REALLY
HELPFUL AND SO GLAD IT IS
EXPANDING TO OTHER
INSTITUTIONS AND I HOPE IT
CAN EXPAND EVEN MORE AND TO
UNDERGRADUATES, POSTDOCS, IT
CAN EXPAND EVEN MORE.
THANK YOU FOR THIS WORK.
>> THANK YOU GINA FOR MAKING
THIS IMPORTANT FUNDING
OPPORTUNITY THAT WE ARE ABLE
TO PROVIDE. YOU CERTAINLY ARE
ONE OF OUR HEROES IN MAKING
THESE SORTS OF COMMUNITY
COHORTS SO IMPORTANT.
>> DR. FINKELSTEIN: KEEP IN
MIND THAT THIS IS JUST ONE OF
A TREMENDOUS SUITE OF
PROGRAMS THAT COMES OUT OF
MICHELLE'S OFFICE. MARGUERITE
AND LAUREN ARE RESPONSIBLE
FOR THEM AND OTHERS.
I AM GOING TO SAVE THE VOTE
TO THE END.
---
THE NEXT CONCEPT IS ABOUT THE
SUDDEN DEATH IN YOUNG
REGISTRY. VICKY IS GOING TO
TALK MORE ABOUT THIS THE
PROGRAM DIRECTOR AND A
CHANNEL SYNOPSIS CLUSTER.
THERE SHE IS.
>> I'M SHARING MY SCREEN
HERE.
>> DR. FINKELSTEIN: OKAY.
>> THANK YOU BOB. SO THE
SUDDEN DEATH IN YOUNG CASE
REGISTRY IS A RENEWAL OF A
PROGRAM THAT THE NINDS HAS
BEEN SUPPORTING SINCE 2015
AND TO GIVE YOU SOME
BACKGROUND THIS IS THE ONLY
POPULATION-BASED SURVEILLANCE
REGISTRY FOR SUDDEN DEATH IN
THE UNITED STATES AND THE
CASE ASCERTAINMENT IS CARRIED
OUT BY STATE, LOCAL PUBLIC
HEALTH OFFICES WORKING
TOGETHER WITH CORNERS OR
MEDICAL EXAMINERS AND WE ARE
CURRENTLY FUNDING 13 STATES
OR JURISDICTIONS MANY
TYPICALLY COUNTIES OR REGIONS
OF THE STATE, IF IT IS NOT
THE ENTIRE STATE.
AND THE REGISTER UTILIZES THE
EXISTING SUDDEN, UNEXPECTED
INFANT DEATH INFRASTRUCTURE
THAT HAS BEEN PUT IN PLACE
AND RUN THROUGH THE CDC AS
WELL AS STATE/CHILD DEATH
REVIEW PANELS AND THEY
COLLECT BIO SPECIMENS AND
INFORMATION ABOUT MEDICAL
HISTORY OF THE CHILD AND THE
CAUSE AND MANNER OF DEATH OF
SUDDEN DEATH UP UNTIL THE AGE
20, DEPENDING ON THE STATE.
---
THE GOALS ARE TO UNDERSTAND
THE CAUSES AND RISKS OF
SUDDEN DEATH, SPECIFICALLY OF
INTEREST TO NINDS 'S SUDDEN
UNEXPECTED DEATH IN EPILEPSY
OR SUDEP, ALTHOUGH THERE ARE
ADDITIONAL NEUROLOGICAL
CAUSES THAT ARE COLLECTED IN
THESE CASES AND FOR NHLBI,
THEY'RE INTERESTED IN
COLLECTING CASES OF SUDDEN
CARDIAC DEATH.
---
ONE OF THE GOALS IS TO
PROVIDE BIOSPECIMENS AND DATA
TO INVESTIGATORS FOR
ADDITIONAL RESEARCH, AND
SUPPORT APPLICATIONS COMING
INTO UTILIZE THE DATA IN THE
REGISTRY AS WELL AS IN THE
BIO SPECIMENS AND MOST
IMPORTANTLY TO INFORM WAYS TO
PREVENT THESE DEATHS IN THE
FUTURE.
---
SO TO TALK ABOUT THIS REALLY
UNIQUE INTRA-AGENCY
COLLABORATION, IT'S FUNDED
PRIMARILY BY NHLBI THAT PUTS
INTO AND HAVE MILLION PER
YEAR TO SUPPORT THIS PROGRAM
BY NINDS AND THE >> CINDY
DAVIS:
(CORRECTION) CDC.
WHO ISSUES NOTICES TO THE
PUBLIC HEALTH OFFICES FUNDED
IN THEIR AREA AND THERE IS A
COORDINATING CENTER CURRENTLY
FUNDED AT NPHI IN MICHIGAN
AND A REPOSITORY THAT IS AT
THE UNIVERSITY OF MICHIGAN
AND AN NHLBI-FUNDED RESEARCH
TO SUPPORT ANALYSIS OF SOME
OF THE BUYER SPECIMEN'S AND I
WILL TALK ABOUT THAT IN THE
NEXT SLIDE. IT HAS BEEN A
VERY FRUITFUL AND FUN PROJECT
TO WORK ON TOGETHER WITH MY
COLLEAGUES ACROSS THE
DIFFERENT AGENCIES, , ACROSS
THE PUBLIC HEALTH OFFICES AS
WELL AS THE INVESTIGATORS.
---
AS I SAID THE FUNDING AND
CASE ASCERTAINMENT BEGAN
JANUARY 2015.
AND 4,955 CASES OF BEEN
IDENTIFY TODAY. ABOUT 70% OF
THE CASE THAT WE COLLECTED
OUR CHILDREN THAT ARE LESS
THAN ONE YEAR OF AGE. THE
AVERAGE NUMBER OF SUDEP CASES
FOR YEARS ABOUT 4% OR ABOUT
30 CASES PER YEAR ON AVERAGE.
ONE OF THE INTERESTING THINGS
WE HAVE SEEN IN THE
DAY-TO-DAY-- ANSWERS REALLY
ONLY FROM A COUPLE OF YEARS
OF ANALYSIS AND WE ARE NOW
ANALYZING MORE OF THE DATA--
IS THAT THE OVERALL INCIDENCE
OF SUDEP IS 0.26 CASES PER
100,000 INFANTS AND CHILDREN
WHICH IS HIGHER THAN WAS IN
THE LITERATURE.
AND THE INCIDENCE IS ACTUALLY
HIGHER IN NONWHITES (BLACKS
AND OTHER POPULATIONS) THAN
IN THE WHITE CHILDREN. NHLBI
FUNDED WHOLE GENOME
SEQUENCING ON 211 CONTENTED
VICE PRESIDENT DATA TO BE
DEPOSITED IN THE DBGAP.
PATHOGENIC OR LIKELY
PATHOGENIC PARENTS WERE
IDENTIFIED AND ONLY 6% OF THE
COHORT OF THE SDY COHORT.
EPILEPSY AND CARDIAC DAMAGE
AND VARIANCE ARE ENRICHED IN
THE SDY COHORT.
INCREASING NUMBER OF RARE
EPILEPSY, BUT NOT CARDIAC
VARIANCE, CORRELATES WITH
YOUNGER AGE AT DEATH.
EPAULETS OF JEAN BURTON
APPEARS TO PLAY A ROLE IN THE
PATHOGENESIS OF SUDDEN DEATH
IN THE YOUNG.
AND WE ARE ANALYZING WHETHER
THESE CHILDREN HAD A
DIAGNOSIS OF EPILEPSY AT THE
TIME OF THEIR DEATH AND IN
TALKING WITH SOME OF THE
PUBLIC HEALTH STAFF THAT ARE
PART OF SOME OF THE
JURISDICTIONS THAT ARE
PARTICIPATING, SOME OF THE
CHILDREN DID HAVE A
DIAGNOSIS, AN INITIAL
DIAGNOSIS, BUT MANY OF THE
CHILDREN NEVER RETURNED TO
HER NEUROLOGY SO THEY MAY
HAVE HAD A DIAGNOSIS AND PUT
ON MEDICATION BUT THERE WAS
NO FOLLOW-UP, THE FAMILY DID
NOT FOLLOW-UP.
---
THERE'S ALSO A NUMBER OF
CASES WHERE THE CHILDREN HAD
MUTATIONS IN SCN 1A, A FAIRLY
COMMON RARE GENETIC EPILEPSY
WHICH THEY DID NOT KNOW THAT
THE CHILD HAD THAT SYNDROME.
SO THERE IS REALLY A NEED FOR
ONGOING RESEARCH IN THIS AREA
AND SO WE ARE RECOMMENDING
CONTINUED FUNDING FOR CASE
ASCERTAINMENT, AND WHAT WE
ARE ADDING AND WOULD LIKE TO
ADD IS INCREASED FUNDING TO
EXPAND BIO SPECIMEN
COLLECTION AT THE TIME OF
AUTOPSY INCLUDING BRAIN
COLLECTION UTILIZING THE NIH
NEURAL BIO BANK SO WE CAN
EXPAND SUDEP RESEARCH AND
RESOURCES FOR INVESTIGATORS.
---
AS ANOTHER COMMENT, NINDS IS
CURRENTLY FUNDING ABOUT $9
MILLION IN RESEARCH ON SUDEP
WHICH WE ARE MAKING
SIGNIFICANT PROGRESS BUT
HAVING ACCESS TO BRAIN TISSUE
WOULD BE REALLY QUITE
VALUABLE FOR OUR ONGOING
RESEARCH IN THIS AREA. I
WILL STOP THERE AND I AM
HAPPY TO TAKE ANY QUESTIONS
ANYONE MIGHT HAVE.
>> DR. FINKELSTEIN: THANKS A
LOT VICKY.
ANY QUESTIONS ABOUT THIS?
-- --
OKAY.
I GUESS-- I'M ONLY SEEING IN
MY SCREEN THE COUNCILMEMBERS
WHO HAVE THEIR CAMERAS ON AND
I AM ASSUMING THERE ARE
OTHERS WHO DON'T.
SO, WHY DON'T WE GO AHEAD TO
THE LAST CONCEPT BEING
PRESENTED.
DAN MILLER, THIS IS ONE FROM
THE NIH BLUEPRINT.
>> HELLO EVERYONE. I'M
SHARING.
AM I SHARING THE CORRECT
SCREEN?
>> DR. FINKELSTEIN: NOT
QUITE.
YOU ARE JUST SHARING A
MONDRIAN WALL.
>> IS THAT IN THE RIGHT
FORMAT?
>> NO, IT'S THE WRONG ONE.
>> DR. FINKELSTEIN: NOT THE
FULL SCREEN.
>> IS THAT FULL-SCREEN?
>> DR. FINKELSTEIN: YES, IT
IS KIND OF A BLANK SLIDE.
>> THAT IS ALL THAT IS.
I HAVE ONE SLIDE AND I WILL
FILL IT IN. THANKS EVERYONE.
THIS SHOULD BE FAIRLY BRIEF.
IT IS A CONCEPT DEVELOPED BY
THE NIH BLUEPRINT SINGLE CELL
TEAM TO SUPPORT JOINT
RESEARCH FOR HUMAN SINGLE
CELL OMIX DATA FROM BRAIN AND
BLUEPRINT ICS. I AM NOT
ENTIRELY SURE HOW MUCH
CONCURRENCE WE NEED SINCE IT
IS A PROPOSAL TO SPEND BLUE
PRINT MONEY AND THERE HAVE
BEEN MULTIPLE LEVELS OF
VETTING ON THIS AND THE IC
DIRECTORS OF ALL APPROVED IT,
BUT WE REALLY DO WANT YOU TO
KNOW ABOUT IT. AND WE WOULD
LOVE TO HAVE YOUR INPUT ON IT.
---
SO WE CONDUCTED-- AN ELASTIC
ANALYSIS OF THE MONEY BEING
SPENT AT NIH ON HUMAN SINGLE
CELL OMICS WORK.
YOU CAN SEE THAT IT'S RISING
FAIRLY RAPIDLY.
MAY BE EXPONENTIALLY.
THE BLUE CHUNKS OF THE BARS
ARE WORKING SUPPORTED AT THE
ICS.
SINCE IT IS SENSITIVE HUMAN
BRAIN OMICS WORK, THIS IS
LARGELY BLUEPRINT ICS.
AND THEN THE-- AND LARGELY
DECEASED FOCUSED
EXPERIMENTS-- AND THE
NON-DARK BLUE BOXES ARE WHAT
THE BRAIN INITIATIVE HAS BEEN
SUPPORTING ESSENTIALLY
BUILDING REFERENCE DATA SETS.
AND SO THAT IS PRETTY MUCH
EXCLUSIVELY WHAT WE CAN
CONTROL, THE NEURO- TYPICAL
TISSUE BEING USED.
AND I WANT TO POINT OUT THAT
IN 2022, YOU PROBABLY ARE
AWARE THAT BICAN (PHONETIC)
IS COMING ON BOARD, A BIG
KICKOFF OF THE HUMAN BRAIN
CELL REGISTRY.
IT WOULD ACTUALLY BE BIGGER
THAN THE 2021 BAR JUST BICAN
ALONE. SO THIS IS RISING AND
RISING VERY RAPIDLY.
---
WHAT WE DID SEE IN THE
ANALYSIS IS THAT THERE SEEMS
TO BE A MISSING PIECE IN THIS
ENVIRONMENT, AND EMPHASIS ON
HARMONIZING THE DATA SET FOR
JOINT ANALYSIS AND COMMON
STORAGE AND SHARING OF BOTH
DISEASE AND CONTROL DATA
SETS. I APOLOGIZE, OF
COURSE-- AS TO COME BY AND
THE BLUEPRINT SAW THIS AS AN
OPPORTUNITY FOR MODERATE
AMOUNTS OF BLUEPRINT MONEY TO
MAKE SOMETHING OF A
DIFFERENCE IN THIS MASSIVE
INVESTMENT THAT NIH IS
UNDERTAKING BOTH AT THE IC
LEVEL AND IN THE BRAIN.
---
SO WHAT WE CAME UP WITH IS
ESSENTIALLY AN INITIATIVE
THAT WE HOPE WILL HELP LAUNCH
HARMONIZATION AND JOINT
STORAGE OF BRAIN AND IC DRIVE
TO DATA SETS.
THIS IS NOT IN SEQUENTIAL
ORDER NECESSARILY, BY
LAUNCHING SOME PILOT PROJECTS
THAT SORT OF PILOT THE JOINT
ANALYSIS AND STORAGE OF THESE
DATA SETS. WE WOULD LOVE TO
SUPPLEMENT ONGOING DISEASE
FOCUSED EFFORTS AND ONGOING
BRAIN INITIATIVE EFFORTS TO
INCLUDE THE OTHER DATA SETS
IN THE FINAL ANALYSIS AND WE
WOULD LIKE TO SUPPLEMENT DATA
ARCHIVES TO INGEST THIS JOINT
DATA AND TO HELP US HARMONIZE
AND SHARE IT AS HARMONIZED
DATA SETS.
---
WE WANT TO CONVENE THE
AWARDEES OF THESE PUBLIC
PROJECTS AND BRAIN-
INITIATIVE FUNDED
INVESTIGATORS AS WELL AS
DECEASED FOCUS PEOPLE INTO A
WORKSHOP AND HEAR BACK ABOUT
WHAT SUCCESSES ARE OF THIS
KIND OF HARMONIZATION EFFORT
AND JOINT ANALYSIS EFFORT BUT
ALSO WHAT THE CHALLENGES ARE.
THE KIND OF HARMONIZATION OF
DATA THAT WAS NEVER BUILT TO
BE UNLESS TOGETHER
UNFORTUNATELY. AND ESTABLISH
SOME COMMON DATA STANDARDS.
---
AND FINALLY WE WOULD LIKE TO
FOLLOW THAT UP WITH SOME
LARGER PHASE THAT FUNDS
JOINT ANALYSIS OF BRAIN AND
IC DATA SETS BASED ON LESSONS
LEARNED FROM THE WORKSHOP,
AND USING THE STANDARD THAT
WE GET ADVICE FROM THE
WORKSHOP; WE ENVISION THIS AS
AN INITIATIVE THAT IS NOT
HUGE BUT GETS IS GOING
TOWARDS PULLING THESE DATA
SETS TOGETHER.
---
I WILL BE HAPPY-- I NEED TO
ACKNOWLEDGE THE TEAM AND
HIGHLIGHTING THE PEOPLE
REALLY INVOLVED IN PUTTING
THIS TOGETHER. I AM HAPPY TO
STOP SHARING AND TAKE
QUESTIONS.
>> DR. FINKELSTEIN: WAIT A
SECOND THERE WAS A SECOND
SLIDE THERE!
>> I APOLOGIZE.
>> DR. FINKELSTEIN: ANY
QUESTIONS?
CLAUDIA I THINK YOU ARE MUTED.
YOU ARE NOT SHOWN AS MUTED
BUT FOR SOME REASON WE CANNOT
HEAR YOU.
F>> HOW ABOUT NOW?
SORRY ABOUT THAT.
SO THANK YOU.
I AM VERY SUPPORTIVE.
I HAVE A COUPLE OF QUESTIONS.
THE WORKSHOP AND THE FOCUS ON
SORT OF THE OUTPUT OF THIS
ACTIVITY.
THOUGHTS AROUND THE INPUT TO
GET TO THAT OUTPUT, THE
MATERIALS AND APPROACHES YOU
ENVISION THE CAN BE A PART OF
THIS. THERE COULD BE A LOT
OF VARIABILITY AND WE TALK
ABOUT SINGLE CELL OMIC DO YOU
ENVISION THIS MOVING ALSO
INTO THE SPATIAL TRANSCRIPT
BROADLY SPEAKING OR ARE YOU
SPEAKING ORDINARILY WITHIN
THE SPACE?
>> WE ARE THINKING SOMEWHAT
MORE LIMITED NOW IN TERMS OF
OMICS.
INTEGRATING DATA ACROSS
ADDITIONAL MODALITIES HAS
ADDITIONAL COMPLICATIONS OF
COURSE.
RIGHT NOW WE ARE REALLY
FOCUSING ON THE OMICS DATA.
BUT WITH YOUR ADVICE WE CAN
EXPAND THE PROJECT TO MORE
THAN WHAT WE ARE THINKING
RIGHT NOW.
>>
>> DR. FINKELSTEIN: TOM?
>> THIS IS GREAT.
AS SOMEBODY WHO IS IN THE
FIELD IN CERTAIN AREAS IT HAS
BEEN HARD TO UNDERSTAND HOW
TO INTERFACE WITH OTHER
INVESTIGATORS SO THIS IS A
GREAT FIRST STEP.
I LIKE THE FACT THAT IT IS AN
EASY TEAR (CORRECTION) TIER
TO HIT.
YOU NOT IMPOSING A LOT. THE
HARMONIZATION ATTEMPTED TO
ALIGN DATA SETS FOR
INTERPRETABILITY ACROSS
PLATFORMS BUT ONE POSSIBILITY
GOING FORWARD IS LOOKING TO
WORK SOMETHING THAT CAN BE
SEARCHABLE SO YOU CAN PULL
OUT A GENE OF INTEREST OR
GENES WITH A PARTICULAR
DIFFERENTIAL REGULATION OR
SIGNIFICANCE ACROSS DATA
SETS. THAT WOULD BE REALLY
INTERESTING AND IT IS A
PROBLEM IN SINGLE CELL,
BECAUSE THE DEPTH OF
SEQUENCING IS LOWER AND
PEOPLE OFTEN REPORT
LARGE-SCALE TRENDS AND
ONTOLOGY'S AND NOT AS MUCH IN
THE BULK REPORT THEIR TYPE
GENES AND THE STATISTIC THAT
DROVE THEM SO YOU COULD CULL
THEM.
IT MIGHT BE INTERESTING JUST
TO VIEW A LITTLE BIT IN THE
FUTURE, YOU WANT TO BE
ONEROUS NOW BUT TO WHAT THE
FUTURE TOWARDS SEARCHABLE
DATA SETS AND THINGS THAT CAN
BE INTERROGATED.
>> DR. FINKELSTEIN:
COMPLETELY AGREE. THANK YOU.
SOUND LIKE A GREAT IDEA.
IF THERE ARE NO MORE
COMMENTS, COULD SOMEONE PUT
UP THE SLIDE OF THE ISSUES
THAT WERE NOT GOING TO BE
DISCUSSED?
I'M TRYING TO SAVE THE NUMBER
OF MOTIONS.
>> I WANTED TO MAKE ONE POINT
THAT IS OKAY BOB.
>> DR. FINKELSTEIN: SURE.
>> I WANT TO THROW OUT THE
IDEA THAT YOUR POINT CAN BE
APPLIED GENERICALLY NOW IN A
SENSE THAT FROM WHAT WE HEARD
EARLIER, ALL THE DATA IS
GOING TO BE POSTED. SO THE
CHALLENGE NOW IS NOT UP
THERE, THE CHALLENGE IS HOW
TO MAKE SENSE OF IT. NO
MATTER WHAT FIELD YOU ARE IN,
YOUR TALKING ABOUT SINGLE
CELL. BUT ALL OF THE FIELDS
PROBABLY CAN BENEFIT BY
THINKING HARD ABOUT HOW TO DO
KIND OF WHAT YOU SET FOR
THEIR FIELD. YOU CANNOT
REALLY DO THAT BEFORE. YOU
HAD TO PAY FOR ALL THE
POSTING. BUT NOW IT'S ALL
GOING TO BE UP THERE. NOW,
IT'S MUCH MORE SIMPLER IN
HIGHER HARMONIZE AND MAKE
SENSE AND BE ABLE TO COMPARE
AMONG DATABASES THAT
EVERYBODY'S GOING TO BE
THROWING UP SOMEWHERE.
--- THERE ARE GRANTS THAT NIH
PUT OUT TO STIMULATE PEOPLE
TO BUILD METADATA STANDARDS
IN THEIR FIELD SO THAT THE
DATA ONCE POSTED WOULD BE
USEFUL.
THERE ARE STILL A COUPLE MORE
DEADLINES FOR THOSE GRANTS.
BUT IF THERE WAS SOMEBODY IN
THE FIELD THAT WANTED TO DO
THIS, THERE IS MONEY TO SET
IT UP.
YOU HAVE TO GET PEOPLE TO BUY
IN, BUT THERE IS MONEY.
>> DR. FINKELSTEIN: THANKS
WALTER.
OKAY, IF I COULD HAVE A SLIDE
OF THE ISSUES THAT WE WERE
NOT GOING TO DISCUSS UNLESS
THERE WERE QUESTIONS, SO YOU
CAN SEE.
I WON'T READ WHAT IT SAYS
HERE.
THESE ARE JUST REISSUES OF
THINGS WE HAVE DONE BEFORE,
LIKE THE JOINTLY SPONSORED T
32 MECHANISM.
UNLESS THERE ARE QUESTIONS
ABOUT THIS I'M NOT GOING TO
DWELL ON ANY OF THEM. PAUSE
THERE.
OKAY, IN THAT CASE WHY DON'T
YOU TAKE THE SLIDE DOWN. WE
NEED A MOTION. SINCE I DID
NOT HEAR ANY OBJECTIONS TO
ANYTHING, WE NEED A MOTION TO
APPROVE ALL THREE THAT WERE
PRESENTED PLUS THE ONES WE
HAVE ON THE SLIDE. I FEEL
THIS IS AN AUCTION.
VOTE IN THE CHAT.
THAT BRINGS US TO THE END OF
THE SESSION WHICH IS A LITTLE
BIT EARLY, ALWAYS NICE.
TOMORROW THE CLOSED SESSION
WILL START AT THE USUAL 1
O'CLOCK.
I ALSO THINK THAT IT'S
POSSIBLE-- I THINK THAT MIGHT
ACTUALLY END A LITTLE EARLY
TOO, BECAUSE WE HAVE A LITTLE
LESS ON THE AGENDA THAT YOU
NEVER CAN TELL.
GREAT TO SEE EVERYBODY AND
SEE YOU IN LESS THAN 24
HOURS.
GREAT TO SEE EVERYBODY.
BYE.