OKAY.
GOOD MORNING, EVERYONE.
WELCOME TO DAY TWO OF THE NIH
DIRECTOR'S ADVISORY COMMITTEE
MEETING.
AND WITHOUT FURTHER ADO, I THINK
WE WILL MARCH RIGHT INTO OUR
AGENDA FOR EVERYONE.
OUR FIRST ITEM IS AN UPDATE ON
THE CANCER MOONSHOT BY NCI
KIMRYN RATHMELL.
LET ME GAVEL US BACK IN.
>> FANTASTIC.
THANK YOU.
IT IS GREAT TO BE HERE.
I'M EXCITED TO GIVE YOU THIS
PRESENTATION ON THE CANCER MO
MOONSHOT EFFORTS.
I WANT TO SAY FIRST THIS
OVERVIEW WILL SHOW YOU HOW WE'VE
LEVERAGED THE CARES ACT FUNDING
AND THE FULL POWER OF MOONSHOT
AS WELL AS A BROAD OVERVIEW OF
THE CANCER PROGRAM.
THIS INFUSION PROVIDED THE AXEL
RANT TO TAKE A READY AND
EXCITING SUBSTRATE AND INDEED
SUCCESSFULLY LAUNCH A TRIP TO
THE MOON AND BACK.
THERE WON'T BE TOO MANY OF THOSE
PUNS.
THE SPACE PROGRAM DEVELOPED A
DEEPER UNDERSTANDING AND
BROADENED OUR CAPABILITIES IN
UNIMAGINABLE WAYS.
WE ARE NOW AS A CANCER INSTITUTE
STRONGLY POSITIONED TO TAKE
CANCER RESEARCH FURTHER AND TAKE
SUCCESSES TO THE AMERICAN PEOPLE
IN WAYS NEVER IMAGINED.
THIS INVESTMENT HAS PAID OFF IN
MASSIVE DIVIDENDS IN A VERY
SHORT PERIOD OF TIME.
WITH SO MUCH MORE ON THIS
HORIZON AS WE'LL DISCUSS.
TO GET STARTED, BY 2016 ROBUST
INVESTMENTS IN THE NATIONAL
CANCER INSTITUTE AND BRAZIL
YANTD WORK ACROSS THE CANCER
COMMUNITY HAD PRODUCED
EXTRAORDINARY EXPERIENCES THAT
CHANGED THE OUTLOOK FOR MILLIONS
OF PEOPLE AT RISK FOR OR LIVING
WITH CANCER IN THE UNITED
STATES.
THE FIRST TARGETED THERAPIES
WERE EMERGING, BRINGING
MUCH-NEEDED AND MORE SELECTIVE
OPTIONS COMPARED TO CHEMOTHERAPY
AND RADIATION.
IMMUNOTHERAPY WAS JUST IN OUR
IMAGINATION AND JUST STARTING TO
APPEAR.
SO MANY OF THE MILLIONS OF
PEOPLE DIAGNOSED ADVANCES WERE
NOT COMING FAST ENOUGH TO GIVE
THEM HOPE AND IMPROVE QUALITY OF
LIFE.
THANKS FOR THE BODY OF EXPERTISE
TO FULFILL THIS PROMISE.
THE CRITICAL INFRASTRUCTURE
FUNDED BY NCI JUST NEEDED THE
RIGHT SPARK TO WORK IN BIGGER
AND BOLDER WAYS.
THE CANCER MOONSHOT WAS THE SHOT
THAT PRESIDENT OBAMA GAVE US A
DECADES WORTH OF PROGRESS IN
JUST FIVE YEARS AND PRESIDENT
BIDEN BROAD BROADER COLLECTIVE
ACTION TO THE FIELD.
THE CANCER MOONSHOT IS A
TRANSFORMATIONAL STORY DPEARNS
SCIENCE, ROCKET TO THE FINAL
FRONTIER WHERE WE END CANCER AS
WE KNOW IT.
I PLAN TO FOCUS ON THAT STORY
AND HOW IT SHAPED INCREDIBLE
ADVANCES AND PROGRESS FOR TODAY.
THE WAY THIS ALL STARTED WITH A
RAPID ACCELERATION WAS TO BRING
TOGETHER A PANEL OF EXPERTS AND
ASK THEM WHERE CRITICAL GAPS LIE
THAT COULD BE ADDRESSED THROUGH
AN INFUSION OF FUNDING.
THIS GROUP, A BLUE RIBBON PANEL
BROUGHT FORWARD THREE BROAD
GOALS.
NUMBER ONE, GENERATE NEW
KNOWLEDGE.
THAT WAS OUR CORE, WHEN WE THINK
ABOUT OUR FOUNDATION IT IS
GENERATING KNOWLEDGE THAT
ADVANCES CANCER.
THAT WAS TRULY ACCELERATED.
THE SECOND BROAD GOAL WAS
ENHANCING COLLABORATION.
YOU WILL SEE A NUMBER OF WAYS
THAT WAS REALIZED.
AND IN 2016 THIS WAS A TIME WHEN
DATA WAS REALLY COMING TO THE
FORE AND IMPROVING DATA SHARING.
I WON'T WALK THROUGH ALL OF THE
RECOMMENDATIONS HERE BECAUSE I'M
GOING TO TAKE YOU THROUGH EACH
OF THEM ONE BY ONE.
WHAT YOU'LL SEE IS PANEL OF 10
RECOMMENDATIONS THAT ALLOWED US
TO MOVE THE FIELD IN A WAY THAT
IS TRULY TRANSFORMATIVE.
THIS IS WHAT THE MOONSHOT LOOKED
LIKE FINANCIALLY.
IN 2017, THERE WAS AN INFUSION
OF $300 MILLION LIKE THE
ACCELERATION AS YOU GAIN
LIFTOFF, THERE WAS AN INCREASE
TO $400 MILLION IN 2019 AS THE
PROGRAM STABILIZE THE FUNDING
WAS, AS YOU CAN SEE, CLOSE TO
$200 MILLION OVER THE NEXT FOUR
YEARS AND THEN COMPLETED AND WAS
COMPLETED IN 2024.
ONE OF THE ADVANTAGES OF THIS
FUNDING IS IT OFFERED MULTIYEAR
AUTHORITY, ALLOWED US TO PLAN
FOR PROGRAMS THAT MIGHT TAKE
ADDITIONAL TIME.
SO WE CONTINUE TO HAVE SOME
FUNDED MOONSHOT PROGRAMS.
THAT WILL CONTINUE PROBABLY FOR
ABOUT THE NEXT YEAR BEFORE THE
FULL EXTENT OF THOSE FUNDS ARE
COMPLETED.
BUT IT ALLOWED US TO THINK MORE
LONGITUDINALLY FOR SOME OF OUR
PROJECTS.
THE MOONSHOT, I'M GOING TO TALK
THROUGH THE WORK THAT HAPPENED
IN THOSE FIRST FEW YEARS OF THE
MOONSHOT, BUT THEN WITH THE WORK
THAT CAME BROUGHT US TO
PRESIDENT BIDEN AND FIRST LADY
JILL BIDEN'S CANCER MOONSHOT.
THIS IS A REAL TRANSFORMATION IN
MENTALITY.
ASKING US TO SET REAL GOALS.
I'M GOING TO TALK ABOUT WHERE WE
HAD ADVANCES IN SCIENCE,
ADVANCES IN CLINICAL CARE,
ADVANCES IN PREVENTION AS WELL
AS TREATMENT.
THAT SET THE STAGE WHERE WE WERE
READY TO BE ABLE TO SET REAL
GOALS.
THESE GOALS REDUCING U.S. CANCER
MORTALITY BY 50% BY 2047 AND
IMPROVE THE EXPERIENCE OF PEOPLE
AND THEIR FEAMGS LIVING WITH AND
SURVIVING CANCER.
-- FAMILIES LIVING WITH AND
SURVIVING CANCER.
THEY SPEAK TO OUR FOUNDATION.
WHAT WE ARE HERE TO DO IS REDUCE
CANCER MORTALITY, REDUCE THE
BURDEN OF CANCER OF PEOPLE IN
THE UNITED STATES.
THE ADVANCES IN THE LAST 30
YEARS FROM RESEARCH AND
DISCOVERY SCIENCE, NEW THERAPIES
HAVE ALREADY REDUCED CANCER
MORTALITY BY 30% OVER THE LAST
30 YEARS.
WE DO HAVE TO DO MORE TO ACHIEVE
50% IN 25 YEARS.
AND IT GETS HARDER AS WE REALLY
TAKE THESE FINAL STEPS IN MAKING
CANCER A DISEASE THAT WE CAN
CONTROL.
BUT WE HAVE NEW TOOLS WE'LL
DESCRIBE HERE IN A MINUTE THAT
WILL TAKE US THERE.
THE OTHER PART OF IMPROVING THE
EXPERIENCE OF PEOPLE AND THEIR
FAMILIES LIVING WITH AND
SURVIVING CANCER ALSO SPEAKS TO
THE TRANSFORMATION THAT HAPPENED
IN THE FIRST FIVE YEARS AROUND
ENGAGING THE PUBLIC, TAKING
PATIENT EXPERIENCE INTO
PERSPECTIVE AND REALLY WHAT WE
AIM TO DO, WHICH IS TO MAKE THIS
A BETTER EXPERIENCE FOR PEOPLE.
THE OTHER REALLY IMPORTANT PART
OF THE REIGNITED CANCER MOON
SHOT WAS THE FORMATION OF THE
CANCER CABINET AND THE
EXPECTATION THAT ALL OF
GOVERNMENT WOULD BE INVOLVED IN
ADDRESSING THIS.
THIS IS NOT AN NCI ONLY PROBLEM.
CANCER TOUCHES LITERALLY EVERY
AMERICAN IN ONE WAY OR ANOTHER.
AND SO IT IS A PROBLEM THAT IS
REALLY REQUIRING THE FULL
ATTENTION OF THE GOVERNMENT.
THE WHOLE OF GOVERNMENT TO
UNLEASH ALL OF OUR POWER AND
JURISDICTIONS IS AN OPEN DOOR
THAT ALLOWED US TO ACCELERATE
EVEN FURTHER.
THE NEXT SLIDE JUST SHOWS SOME
OF THE AGENCIES THAT WE NOW VERY
ACTIVELY PARTNER WITH.
WE HAD ALWAYS HAD GOOD
COLLABORATIONS WITH THESE
ORGANIZATIONS, BUT WITH THE
CREATION OF THE CANCER CABINET
AND THE EXPECTATION WE ARE ALL
ON ONE TEAM IT HAS REALLY
ALLOWED TREMENDOUS RESURGENCE IN
RESEARCH, FOR EXAMPLE, THAT
UTILIZES THE DEPARTMENT OF
DEFENSE AND THE VA EXPERTISE,
LEVERAGES WHAT THE DEPARTMENT OF
ENERGY HAS TO OFFER, PARTNERS
WITH THE CDC WHERE CANCERS ARE
EMERGING IN DIFFERENT WAYS.
UNDERSTANDING HOW THE
ENVIRONMENT CONTRIBUTES TO
CANCER WITH PARTNERSHIPS WITH
THE EPA AND I WILL SPEAK WWF
SPECIFIC WAYS WORK WITH THE FDA
ACCELERATED THE WORK THAT WE DO.
NOW I'M GOING TO WALK THROUGH
THOSE BLUE RIBBON PANEL
RECOMMENDATIONS AND GIVE YOU A
HIGH LEVEL SUMMARY WITH SOME
EXAMPLES OF HOW THESE
RECOMMENDATIONS WERE REALIZED IN
THIS REALLY VERY SHORT PERIOD OF
TIME.
THIS WAS JUST AN EYE CHART.
JUST TO LET YOU KNOW I'M SHOWING
YOU SOME EXAMPLES, SOME PIECES
THAT ARE REPRESENTATIVE.
THIS IS A SLIDE THAT WE LIKE TO
SHOW PARTLY SO WE KEEP TRACK OF
THE MOVING PIECES S PROJECTS,
PROGRAMS AND COORDINATING
CENTERS AND PIECES TO THE
MOONSHOT.
EACH IS LINKED BACK TO THE BLUE
RIBBON PANEL RECOMMENDATIONS.
SO LET'S JUST START WITH NUMBER
ONE.
I WILL SAY FIRST THESE ARE NOT
RANKED BY PRIORITY.
THEY ARE ALL IMPORTANT AND IS
THERE ARE CROSS CUTTING PIECES
IN EACH PART OF THESE BLUE
RIBBON PANEL RECOMMENDATIONS.
FIRST, ESTABLISHING A NETWORK
FOR DIRECT PATIENT ENGAGEMENT.
THIS IS A REAL TRANSITION TO
ENCOURAGE AND DEMAND THAT WE
WORK VERY CLOSELY WITH PATIENTS
TO HAVE THEIR OPINIONS ENGAGED
IN THE SCIENTIFIC WORK THAT WE
DO.
THAT HAS BEEN HARDER TO DO IN
THE PAST, BUT IS NOW VERY MUCH A
PART OF OUR NORMAL CULTURAL
ECOSYSTEM.
I KNOW YOU HEARD A LOT ABOUT
THAT YESTERDAY IN THE VARIOUS
PRESENTATIONS ABOUT HOW MUCH
PATIENTS AND ADVOCATE INPUT CAN
REALLY INFORM, ENRICH AND
ACCELERATE OUR SCIENCE.
THIS IS JUST ONE EXAMPLE.
IT IS A PARTICIPANT ENGAGEMENT
AND CANCER GENOME SEQUENCING
NETWORK.
PECGS, WE LIKE OUR ACRONYMS.
A NETWORK TO DEVELOP
PARTNERSHIPS WITH PATIENTS
ADVANCING STUDIES PARTICULARLY
IN RARE PATIENT POPULATIONS
BECAUSE OF THE DISEASE OR
SEGMENT OF THE POPULATION AND TO
UNDERSTAND HOW THOSE WERE
RECEIVE AND INTERPRETED BY
PATIENTS SO WE COULD DETERMINE
BEST PRACTICES FOR HOW WE
ENGAGE.
IF YOU THINK BACK TO 2015, 2016
EN, WHEN THIS WAS ALL STARTED IT
WAS FAIRLY UNCOMMON TO DO A
LARGE PANEL GENETIC TEST ON A
PATIENT'S TUMOR.
IT WAS ALMOST UNHEARD OF TO DO
WHOLE GENOME SEQUENCING.
THOSE ARE THINGS WE DO ROUTINELY
TODAY AND WE WOULD NOT HAVE BEEN
READY TO OFFER THOSE, TO
UNDERSTAND THE BEST PRACTICES TO
BE ABLE TO INTERFACE WITH
PATIENTS HAD WE NOT TAKEN THIS
STEP VERY EARLY ON.
THIS IS ANOTHER EXAMPLE OF
NETWORKS FOR DIRECT PATIENT
ENGAGEMENT.
MY PART, MY PEDIATRIC AND ADULT
RARE TUMOR NETWORK BRINGS
PATIENTS TOGETHER WHO EXPERIENCE
RARE TUMORS.
MUCH OF OUR ACTIVITY BY
NECESSITY HAS FOCUSED ON THE
MORE COMMON TUMORS, BUT MANY
TUMORS OCCUR IN FEWER THAN
40,000 PATIENTS PER YEAR, BEING
ABLE TO CONNECT THESE PATIENTS
SO THEY CAN IDENTIFY THE RIGHT
PROVIDERS, THE RIGHT TREATMENTS
TO LEARN WHAT THE FOUNDATIONAL
COMPONENTS OF THEIR DISEASES ARE
IS REALLY ESSENTIAL AND REQUIRES
THAT LEVEL OF COMMUNITY OUTREACH
AND HAS BEEN A VERY SUCCESSFUL
PROGRAM.
ANOTHER PROGRAM SIMILAR TO MY
PART FOCUS ON BUILDING A NETWORK
FOR RARE BRAIN AND SPINE TUMORS.
SO NCI CONNECT.
IT WAS PARTNERSHIPS TO BRING
TOGETHER PATIENTS AND PROVIDERS
AROUND RARE BRAIN AND SPINE
TUMOR NETWORKS AND HAS LED TO
SOME TREMENDOUS DISCOVERIES, ONE
OF WHICH I WILL TALK ABOUT LATER
IN THE TALK.
THE SECOND RECOMMENDATION WAS
CREATING TRANSLATIONAL SCIENCE
NETWORK DEVOTED EXCLUSIVELY TO
IMMUNOTHERAPY.
AGAIN, IF YOU THINK BACK TO
2015, THE FIRST IMMUNOTHERAPIES
WERE BEING APPROVED.
THE FIRST IMMUNOTHERAPY FOR
MELANOMA WAS THE END OF 2017.
SMALL LUNG CELL CANCER CAME OUT
IN 2015.
WE KNEW THERE WAS A GLIMMER OF
HOPE.
IT WAS PRESCIENT TO SAY WE
NEEDED NETWORKS TO TACKLE
IMMUNOTHERAPY.
THE CANCER MOONSHOT SUPPORTED A
VARIETY OF VERY FOCUSED
NETWORKS.
I HAVE SOME EXAMPLES.
IMMUNO-ONCOLOGY TRANSLATION
NETWORK, CELLULAR MONITORING
LOOKING AT THE MICROENVIRONMENT,
A NETWORK THAT USES CANINE MODEL
SYSTEMS TO UNDERSTAND HOW
IMMUNOTHERAPY COULD WORK BEST.
THESE BROUGHT LIKE-MINDED
INVESTIGATORS TOGETHER TO
ELIMINATE REDUNDANCY, BE ABLE TO
WORK TOGETHER TO MAKE THE REAL
ADVANCES THAT WOULD TAKE
IMMUNOTHERAPY TO THE PLACE WHERE
IT IS TODAY.
THIS IS JUST SOME NUMBERS.
IN THE TIME SINCE THE INITIATION
OF THE MOONSHOT WE HAVE SEEN 85
ANTI-P.D-1 ANTI-FDA APPROVALS.
THAT IS FOR 22 IN NONSMALL CELL
LUNG CANCER.
SO THESE DRUGS ARE FINDING NEW
WAYS TO ADDRESS THESE CANCERS
IN -- THAT CAN MOVE US CLOSER
AND CLOSER TO TREATING PATIENTS
AT THE EARLY STAGES SO WE NEVER
HAVE TO GET TO THE LATER STAGES.
WE HAVE SEEN 39 NON-PD-1/L1
IMMUNO ONCOLOGY FDA APPROVALS IN
16 CANCER TYPES.
13 IN NONHODGKINS LYMPHOMA AND
T-CELLS WERE ON THE VERGE OF
SEEING VACCINES FOR CANCER
APPROVED.
SO THIS AREA HAS JUST EXPLODED.
I TALKED BEFORE ABOUT THE
NETWORK FOR IMMUNOTHERAPY IN
FOCUSED AREAS.
IT IS EXCITING TO SEE LARGE
PROGRAMS NOT DIRECTED BY THE
NCI, SITAC, A VERY SMALL
ORGANIZATION AND TODAY LARGE
NUMBERS.
WE ARE A PARTNERING INSTITUTION
SO IT TAKES THE ENGAGEMENT
ACROSS MANY DIFFERENT PARTS TO
BE ABLE TO MAKE THE INROADS WE
HAVE SEEN IN IMMUNOTHERAPY
TODAY.
THIS HAS ALSO ALLOWED US TO TAKE
THESE NETWORKS TO UNDERSERVED
POPULATIONS.
A REALLY NOTABLE ONE IS
PEDIATRIC CANCERS.
THEY ARE VERY RARE TUMORS AND
MANY PEDIATRIC CANCERS, BUT NOT
MANY IN NUMB.
THE PEDIATRIC IMMUNOTHERAPY
NETWORK BROUGHT TO OUR MOST
DIFFICULT CANCERS.
THIS IS THE STORY THAT I
REFERENCED.
JUST PUBLISHED LAST MONTH IN
"NATURE."
BOTH SPEAKS TO BRINGING TOGETHER
POPULATIONS AND THE NETWORKS OF
IMMUNO THERAPY TO BRING NOVEL
IMMUNO THERAPY TO RARE PEDIATRIC
TUMORS.
DIPG IS A VERY AGGRESSIVE
PEDIATRIC BRAIN TUMOR THAT
OCCURS IN KIDS IN THE LATE
ELEMENTARY SCHOOL AGE RANGE.
MEDIAN SURVIVAL 11 MONTHS.
10% OF PATIENTS SURVIVE AFTER
DIAGNOSIS.
THIS HAS ALWAYS BEEN ONE OF THE
DREADED DISEASE FOR KIDS WITH
BRAIN TUMORS.
THIS CAR T RF CELL THERAPY
FUNDED BY THE NCI PUSH LISHED ON
A DECENT SIZE NUMBER OF PATIENTS
FOR SUCH A RARE DISEASE AND
SHOWED THERAPY SHRANK TUMORS FOR
A DURABLE PERIOD OF TIME IN
THESE PATIENTS.
THIS IS TREMENDOUSLY EXCITING
AND A REAL ADVANCE FOR AN
IMPORTANT GROUP OF PATIENTS.
THE THIRD RECOMMENDATION WAS TO
DEVELOP WAYS TO OVERCOME
CANCER'S RESISTANCE TO THERAPY.
WE KNOW THAT THIS IS WHERE WE
ULTIMATELY LOSE PATIENTS IS WHEN
OUR THERAPIES START TO FAIL.
WE CAN LEARN AS WE HAVE MORE
OPTIONS OF WHAT WE CAN COMBINE,
COMBINING DIFFERENT CANCER DRUGS
TOGETHER, UNDERSTANDING HOW WE
PUT THOSE PIECES TOGETHER,
KEEPING CANCER DRUGS INSIDE
CELLS.
CELLS HAVE GOOD WAYS OF
EXTRUDING THEM.
ERASING REVERSIBLE
MODIFICATIONS.
ALTERING THE TUMOR
MICROENVIRONMENT AND TESTING
THOSE COMBINATIONS.
THIS HAPPENS IN THE LABORATORY.
THIS IS PARTLY UNDERSTANDING THE
BASIC SCIENCE, THAT UNDERLIES
HOW WE CAN OVERCOME RESISTANCE.
THIS HAPPENS IN THE CLINIC.
AN IMPORTANT PART OF THE CANCER
MOONSHOT WAS CREATED IF NRK FORM
LARRY A PUBLIC-PRIVATE
PARTNERSHIP THAT ALLOWS FOR
INVESTIGATORS TO IDENTIFY
COMBINATIONS OF DRUGS AND OFFER
THEM IN CLINICAL TRIALS TO
PATIENTS.
IT REQUIRED PARENT --
PARTNERSHIPS WITH AGENCIES AND
FO
FORMULARY OF 35 AGENTS TO BE
USED ACROSS A WIDE VARIETY OF
SITES.
IT HAS TAKEN US INTO THE PATIENT
POPULATION.
THIS IS JUST ONE EXAMPLE OF A
STUDY THAT REALLY CAPITALIZED ON
HOW DO WE USE OUR CURRENT
KNOWLEDGE IN COMBINING DRUGS TO
OVERCOME RESISTANCE.
THIS IS THE VIPOR STUDY.
THE PILOT WAS DONE AT NCI.
IF WE UNDERSTOOD THE GENETIC
COMPOSITION OF THE TUMOR WE
COULD CREATE THE RIGHT COCKTAIL
TO ERADICATE THE CANCER.
THIS IS A STORY WE TOLD IN OUR
ANNUAL REPORT THIS YEAR OF A
PATIENT NAMED JUSTIN WHO HAD
REFRACTORY NONHODGKIN'S
LYMPHOMA, PROGRESSED ON ALL
STANDARD THERAPIES, BUT BASED ON
HIS GENETIC MAKEUP WAS BROUGHT
TO THIS STUDY, RECEIVED THE
COCKTAIL THERAPY AND IS NOW
THREE YEARS OFF THERAPY WITHOUT
ANY EVIDENCE OF DISEASE AND
DOING WELL.
THIS IS THE KIND OF STORY THAT
WE WANT TO TELL.
THIS IS THE KIND OF STORY THAT
COMES FROM EXACTLY FOCUSING ON
WORK OF THAT PARTICULAR MOONSHOT
RECOMMENDATION.
NUMBER FOUR WAS BUILDING A
NATIONAL CANCER DATA ECOSYSTEM,
RECOGNIZING WE HAVE LARGE
AMOUNTS OF DATA.
THEY HAVE TO BE ABLE FOR
REREVIEW, REANALYSIS,
COMBINATION.
CREATION OF THE NCI CANCER
RESEARCH DATA COMMONS BROUGHT
TOGETHER NCI PROJECTS TO BE
ANALYZED BY MORE INVESTIGATORS.
THIS HAS BECOME A MAJOR PART OF
WHAT WE DO, COMBINING THE DATA
COMMONS, CLOUD BASED RESOURCES
AND UNDERLYING INFRASTRUCTURE.
WE HAVE JUST SEVERAL EXAMPLES.
I WILL TALK ABOUT THE CHILD
CANCER DATA INITIATIVE.
THAT IS AN INITIATIVE THAT AIMS
TO LEARN FROM EVERY PEDIATRIC
PATIENT WITH CANCER BY INCLUDING
THEIR DATA FOR REAL TIME
ASSESSMENT.
WE CAN USE THIS DATA FROM REAL
WORLD, KLINE -- CLINICAL TRIALS
AND WELL CURATED DATA.
WE HAVE RECOMMENDATION TO
INTENSIFY RESEARCH ON THE MAJOR
DRIVERS OF CHILDHOOD CANCER.
I ALREADY MENTIONED THAT
PEDIATRIC CANCERS HAVE LONG BEEN
DIFFICULT TO STUDY BECAUSE OF
HOW RARE THEY ARE.
EVERYONE WITHIN RARE TUMORS THE
MUTATIONS CAN BE VERY UNCOMMON.
ONE OF THE INITIATIVES OF THE
CANCER MOONSHOT THAT IS REALLY
EXCITED WAS THE FUSION
ONCOPROTEINS IN CHILDHOOD
CANCERS.
FUSONC2 CONSORTIUM, AIMED TO
CATEGORIZE THE FUSION CREATED IN
CHILDHOOD CANCERS.
FUSION IS TWO GENES THAT ARE
BROUGHT TOGETHER TO CREATE
SOMETHING NEW AND NOVEL WITH A
NEW ACTIVITY THAT IS A CANCER
DRIVER IS MUCH MORE COMMON IN
PEDIATRIC CANCERS THAN ADULT
CANCERS.
THE RECOGNITION OF WHAT THESE
FUSIONS ARE, HOW THEY WORK AND
WHERE WE CAN FIND THEM HAS NOW
LED TO A NEW NETWORK TARGETING
FUSION ONCOPROTEINS IN CHILDHOOD
CANCERS.
THIS IS A TREMENDOUSLY EXCITING
OPPORTUNITY FOR A VARIETY OF SAR
KOCOMB -- SARCOMAS THAT ARE IN
YOUNG CHILDREN.
I TOLD YOU I WOULD MENTION THE
CHILDHOOD CANCER DATA
INITIATIVE.
THIS WAS AN INITIATIVE THAT CAME
IN THE FIRST TRUMP
ADMINISTRATION.
AS I SAID, TO BUILD A COMMUNITY
AROUND CHILDHOOD CANCER CARE AND
RESEARCH DATA AND LEARN FROM
EVERY CHILD.
WITH THE STAR ACT, WHICH WAS
ALSO AN EMERGENCE FROM THE
CANCER MOONSHOT, WE FOLDED IN
MOLECULAR CHARACTERIZATION.
LEARN FROM EVERY PATIENT AND
LEARN FROM THE MOLECULAR
CHARACTERIZATION FROM AS MANY
PATIENTS AS POSSIBLE PROVIDES A
STATE OF THE ARD MOLECULAR
CHARACTERIZATION TO FORM THE
BEST AND MOST APPROPRIATE
TREATMENT.
WE ARE LEARNING NOT ONLY ABOUT
FUSION ONCOPROTEINS BUT OVER
DRIVERS PREVIOUSLY OVERLOOKED.
WE ARE LEARNING ABOUT RISK
FACTORS FOR CHILDREN AND WHY
CHILDREN MIGHT BE AT RISK FOR
CANCER THAT HAD PREVIOUSLY NOT
APPRECIATED.
SO THIS IS A SET OF DATA THAT
GOES INTO THE CANCER RESEARCH
DATA COMMONS THAT WILL CONTINUE
TO FUEL OUR UNDERSTANDING AND
FUTURE THERAPIES FOR PEDIATRIC
CANCERS AND CANCERS IN ADULTS AS
WELL.
NUMBER SIX.
MINE MIZING CANCER TREATMENT'S
DEBILITATING SIDE EFFECTS.
THIS IS TO RECOGNIZE IT IS NOT
JUST GETTING THE PATIENT TO THE
RIGHT CANCER TREATMENT, BUT
TAKING CARE OF THAT PATIENT WELL
FOR LIFE DURING TREATMENT AND
AFTER CANCER.
SO ONE OF THE OUTPUTS OF THIS IS
THE NATIONAL STANDARDS FOR
CANCER SURVIVORSHIP CARE.
THIS IS THE FIRST GOVERNMENTWIDE
SET OF STANDARDS FOR HOW TO TAKE
CARE OF PATIENTS AFTER A CANCER
DIAGNOSIS.
IT PROVIDES THESE STANDARDS TO
SET ESSENTIAL HEALTH SYSTEM
POLICY AND THIS WAS CREATED IN
PARTNERSHIP WITH THE VA AND WAS
JUST ANNOUNCED EARLIER THIS FALL
AND PERCOLATING THROUGH CANCER
CARE ACROSS THE COUNTRY.
ALSO IN MINIMIZING DEBILITATING
SIDE EFFECTS WAS UNDERSTANDING
CANCER TREATMENT'S TOLL
REHABILITATE.
WE HAVE HAD STANDARDIZED --
TOLERA
TOLERABILITY.
SPEAKING TO THE MANDATE TO ASK
OUR PATIENTS WE HAVE TURNED
INCREASINGLY TO PATIENT-REPORTED
OUTCOMES.
SO THIS IS A UO1 CONSORTIUM
CALLED ANALYSIS OF PRO-CTCAE AND
OTHER PATIENT REPORTED OUTCOMES
TO BE ABLE TO MUCH MORE ROBUSTLY
CAPTURE INFORMATION FROM
PATIENTS ABOUT HOW THEY ARE
FEELING ON THE DAY THEY COME TO
SEE THEIR DOCTOR AND HOW THEY
ARE FEELING WHEN THEY ARE HOME
AND HALFWAY IN BETWEEN THEIR
CANCER THERAPY.
THIS ALLOWS ANALYSIS OF BASELINE
SYMPTOMS, POST FACTORS TO
INFLUENCE SIDE EFFECTS.
PHARMACOKINETIC DATA.
THAT IS ALREADY BEGINNING BUT
WILL BE A MUCH BIGGER PART OF
CANCER CARE BECAUSE OF THIS
WORK.
ALSO ON MINIMIZING DEBILITATING
SIDE EFFECTS ANOTHER CONSORTIUM,
IMPROVING THE MANAGEMENT OF
SYMPTOMS DURING AND FOLLOWING
CANCER TREATMENT.
IT CAME OUT TO BE IMPACT.
WAYS WE CAN ENABLE PROVIDERS TO
UTILIZE THE ELECTRONIC HEALTH
RECORD.
HOW WE CAN USE BILINGUAL
ELECTRONIC SYMPTOM MANAGEMENT TO
PROVIDE CARE TO PATIENTS WHO
SPEAK A DIFFERENT LANGUAGE AND
ENHANCING OUR PATIENT REPORTED
OUTCOMES FOR SYMPTOM MANAGEMENT.
NOT JUST DATA COLLECTION BUT
BIDIRECTIONAL CARE IN THE
PATIENT ENVIRONMENT.
NUMBER SEVEN.
WE ARE GETTING THERE.
EXPANDING THE USE OF PROVEN
CANCER PREVENTION AND EARLY
DETECTION STRATEGIES.
THIS IS WHERE WE HAVE HAD REAL
LANDSCAPE SHIFT IN CANCERS,
THINKING ABOUT PREVENTION.
I POINT TO THE UPPER RIGHT HAND
BOX TO POINT OUT PREVENTION AND
SCREENING EFFORTS HAVE AVERTED
4.7 MILLION DEATHS FROM FIVE
CANCER TYPES.
WE PUBLISHED THIS PAPER EARLIER
IN THE YEAR.
THE INVESTMENT IN PREVENTION FAR
OUTWEIGHS THE EXPENSE OF
TREATMENT.
THE MORE WE CAN DO TO PREVENT
CANCER THE BETTER THE ECONOMIC
RETURN.
THE ACCELERATED COLO RECTAL
SCREENING.
WHICH INCORPORATED A NAVIGATOR
WHO IS A START PART OF CANCER
CARE, BUT UTILIZE NAVIGATORS TO
ENABLE PATIENTS TO GET THROUGH
SCREENING AND DIAGNOSE FOR COLON
CANCER.
MUCH FOCUS ON SMOKING CESSATION.
I WANT TO TAKE A MINUTE AND TALK
ABOUT HPV.
THIS WORK EXPANDED FUNDING FOR
HPC, TRIALS THAT ARE TESTING
SINGLE DOSE OF HPV VACCINE TO
SEE IF IT IS SUFFICIENT TO
PREVENT HPV DRIVEN CANCERS
PARTICULARLY CERVICAL CANCER AND
TESTING THE SUBTYPE FOR
INDEPENDENT PATIENT RISK
ASSESSMENT.
I'M GOING TO TALK ABOUT HPV A
LITTLE MORE.
LAST SPRING FALLING ON THE HEELS
OF SUCCESSFUL MOONSHOT FUNDED
EFFORTS TO BRING HPV VACCINATION
TO THE FORE WE ANNOUNCED THE
SHIP TRIAL.
THIS IS OUR LAST MILE TRIAL TO
BRING SCREENING TO PATIENTS WHO
ARE, FOR A VARIETY OF REASONS,
UNABLE TO RECEIVE STANDARD J
GYNECOLOGY CARE.
ALLOWING PATIENTS TO DO THEIR
OWN RISK ASSESSMENT COULD ALLOWS
US TO ERADICATE CERVICAL CANCER
THROUGH A COMBINATION OF HPV
VACCINATION, GOOD SCREENING AND
EFFECTIVE EARLY CARE.
NEXT SLIDE.
NUMBER EIGHT IS MINING PAST
PATIENT DATA TO PREDICT FUTURE
PATIENT OUTCOMES.
WITH THIS VAST AND GROWING ARRAY
OF DATA, THERE ARE THINGS WE CAN
LEARN FROM THIS CAN ACCELERATE
OTHER WORK.
MOLECULAR PROFILING TO PREDICT
RESPONSE TO TREATMENT.
WE HAVE BIOSES MENS ARCHIVED
THAT CAN BE AND HAVE BEEN TESTED
BY NEXT GENERATION SEQUENCING,
TESTED, AND USED COMPUTATIONAL
MODELLING TO UNDERSTAND WHAT
MUTATIONS LED TO RESPONSES TO
THERAPY.
THIS PRECLINICAL WORK COMMITTED
FOR A MORE ADVANCED CLINICAL
TRIAL.
THIS IS ONE EXAMPLE THAT HAS
FUELED MULTIPLE EXCITING
INNOVATIVE CLINICAL TRIALS.
NUMBER NINE IS DEVELOPING A 3-D
CANCER ATLAS.
THIS ONE WAS CLEAR AND WE
UNDERTOOK IT WAS FIRST PUBLISHED
ON LAST YEAR AND THIS YEAR WE
BROUGHT IN A BIGGER COMPLEMENT
OF TUMORS.
WE NEVER THOUGHT THAT WHEN I WAS
TRAINING ABOUT THE VERY DYNAMIC
COMPONENTS OF WHAT'S INSIDE A
TUMOR.
THE IMMUNE CELLS, THE VASCULAR
CELLS, HOW THEY ARE INTERACTING
DYNAMICALLY IN REAL TIME.
LOOKING IN THREE DIMENSIONS AT
THE TUMORS OF WHO IS TOUCHING
WHO GIVES A DIFFERENT VIEW OF
HOW WE SEE A TUMOR.
SO THE NETWORK PROVIDES
THREE-DIMENSIONAL VISHTIZATION
AND CREATING A DATA REPOSITORY
THAT IS AVAILABLE FOR
INVESTIGATORS ALL OVER THE
COUNTRY.
2,000 CASES, 9,000 BIOSPECIMENS
ANALYZED AND AVAILABLE AND
RECENTLY PUBLISHED ON.
IT IS NOT JUST A REPOSITORY OF
IMAGES BUT OF TOOLS TO ALLOW
INVESTIGATORS TO BE ABLE TO DO
THE MODELLING AND INTERSECTION
NETWORK ANALYSIS, TO LOOK AT
THEIR TUMOR, THEIR QUESTION,
THEIR CELLS AND BE ABLE TO
ADDRESS NEW INNOVATIONS THAT
COME FROM UNDERSTANDING THIS
THREE DIMENSIONAL LOOK AT TUMOR.
THIS IS A DIFFERENT WAY TO
LOOKING AT TUMORS TO CANCER
INVESTIGATORS ACROSS THE
COUNTRY.
NUMBER O10 IS DEVELOPING NEW
CANCER TECHNOLOGIES.
THIS IS WHAT WE FEEL IS OUR
BREAD AND BUTTER, BUT HAS WORKED
TO ACCELERATE PARTNERSHIPS WITH
INDUSTRY, BUT TO BRIDGE THE GAP
BETWEEN THE DISCOVERY OF A NEW
TECHNOLOGY TO GET IT TO A PLACE
WHERE IT CAN MOVE INTO THE
CLINICAL DOMAIN.
I HAVE JUST SOME OF THE NEW
TECHNOLOGIES THAT HAVE COME ON
THE SCENE DURING THIS TIME.
CAR T-CELLS, TU FORINFILTRATING,
CRISPR, PRO-T AC, ANTIBODY
CONJUGATES.
I WON'T READ THEM ALL, IMMUNO
THERAPY AND MOLECULAR THERAPY ON
A DIFFERENT LEVEL TODAY.
SO HAVING BEEN THROUGH THE 10
RECOMMENDATIONS, I WANT TO JUST
TELL YOU A FEW MORE THINGS ABOUT
THE MOONSHOT AND WHAT THE
PROGRAM DID FOR US.
PART OF IT WAS LOOKING AT OUR
WORKFORCE AND USING THOSE FUNDS
TO ENRICH AND DIVERSIFY OUR
WORKFORCE.
THE CANCER MOONSHOT PROGRAM WAS
REIGNITED IN THE CANCER MOONSHOT
TO BRING YOUNG, TALENTED
INVESTIGATORS TO ADVANCE CANCER
SCIENCE AND THOUGHT APPROACH.
WE ARE IN OUR THIRD CLASS OF
MOONSHOT SCHOLARS.
I WILL INTRODUCE YOU TO THREE OF
THEM.
THESE THREE ARE FROM OUR MOST
RECENT CLASS.
THEY ARE ALL INVESTIGATING
EXCITING COMPONENTS FROM REALLY
BASIC SCIENCE TO CLINICALLY
BASED RESEARCH SUCH AS THE
IMAGING STUDY IN THE MIDDLE AND
PARTNERSHIPS IN THE COMMUNITY.
COMING AT CANCER FROM THE RANGE
OF TYPES OF QUESTIONS THAT ARE
IMPORTANT.
THESE PEOPLE ARE REALLY OUR
FUTURE.
HOW DO WE ASSESS THE IMPACT OF
THIS PROGRAM?
SO WE -- I THINK THE IMPACT IS
IN WHAT I HAVE JUST TOLD YOU.
HOW WE THINK DIFFERENTLY ABOUT
HOW WE ENGAGE WITH THE
COMMUNITY.
HOW WE THINK DIFFERENTLY ABOUT
WHAT WE DO IN TERMS OF
COLLABORATIONS AND HOW WE
ADVANCE INNOVATION IN A MUCH
MORE NETWORKED WAY AND PUT THE
DATA OUT THERE FOR EVERYONE TO
CAPITALIZE ON.
WE CAN ALSO QUANTIFY THIS IN
PUBLICATIONS.
AT LEAST 3,400 PUBLICATIONS.
IT BECOMES INCREASINGLY
DIFFERENT TO ASSIGN PUBLICATIONS
AS THIS PERMEATES AND EVERYONE
IS DOING CANCER MOONSHOT.
89 CLINICAL TRIALS, 78 PATENT
FILINGS.
IT IS IMPORTANT TO KNOW IN SOME
WAYS THAT THE CANCER MOONSHOT
WAS DIFFERENT THAN SOME OF THE
WORK THAT WE TRADITIONALLY DO.
OUR NORMAL GRANT MECHANISM IS
THE RO1.
WE DID FUND RO1s THROUGH THE
CANCER MOONSHOT, IT WAS
DISPROPORTIONALLY SKEWED TO UO1,
PROJECT BASED AND MOVED BACK
INTO THE INVESTIGATOR INITIATED
PARTSES OF THE PROGRAM.
-- PARTS OF THE PROGRAM.
THE NEXT COUPLE OF SLIDES SHOW
WHERE THE IMPACT HAPPENED.
NOT JUST THE DISCOVERIES THAT
WERE MADE, BUT THE RESEARCH THAT
WAS HAPPENING.
WHAT YOU CAN SEE HERE IS OF THE
FUNDED WORK, SO OVER 4,000
PROJECTS THAT WERE FUNDED
THROUGH THE MOONSHOT, WHEN WE
LOOKED AT NEW VS. ONGOING
COLLABORATIONS WE SAW THE
MAJORITY OF COLLABORATIONS WERE
ACTUALLY NEW.
PEOPLE WERE BROADENING THEIR
HORIZONS TO TAKE THEIR WORK IN
NEW DIRECTIONS.
AS WE LOOKED AT THE OFFSHOOTS OF
THAT WORK, THOSE COLLABORATIONS
CONTINUE AND CONTINUE TO BE IN
SOME CASES FUNDED THROUGH
ONGOING MOONSHOT WORK, BUT
CONTINUE WITHOUT MOONSHOT
FUNDING AS WELL.
SO WE'RE REALLY EXCITED ABOUT
WHAT THIS HAS DONE TO OUR
COMMUNITY OF INVESTIGATORS.
WE'RE ALSO REALLY EXCITED ABOUT
HOW MANY YOUNG PEOPLE TOOK
ADVANTAGE OF THE MOONSHOT.
THIS WAS NOT JUST AN INFUSION OF
FUNDING THAT SUBJECTED
ESTABLISHED INVESTIGATORS.
AS YOU CAN SEE HERE ABOUT A
THIRD OF OUR INVESTIGATORS WERE
FIRST-TIME GRANT RECIPIENTS.
41 OF WHOM ARE EARLY STAGE
INVESTIGATORS.
YOUNG INVESTIGATORS SERVING FOR
THE FIRST TIME AS A PI ON A
MOONSHOT GRANT.
SO THIS ALSO WAS AN INFUSION OF
PEOPLE INTO OUR ECOSYSTEM.
THIS ALSO SAW OVER THIS TIME
PERIOD A REAL DRAMATIC CHANGE IN
DATA ACCESS AND DATA SHARING.
I WILL SAY THIS ALSO CAME WHEN
MANY OTHER THINGS WERE CHANGING
ABOUT EXPECTATIONS AROUND DATA
SHARING, INCLUDING DEMANDS BY
JOURNALS.
THERE WAS A DEMAND ON OUR PART
AS WELL THAT DATA BE MADE
AVAILABLE EARLY AND IN ITS
ENTIRETY FOR MOONSHOT PROJECTS.
AND YOU CAN SEE FROM THE
REDLINE, THE NUMBER OF PROJECTS
FOR WHICH ALL DATA WAS AVAILABLE
WAS VERY LOW AND MADE A MARKED
INCREASE OUT TO 2023, WHERE WE
HAVE THIS DATA FOR.
THE STUDIES THAT PROVIDED NO
DATA, WHICH WAS A COMMON EVENT
IN 2017 IS NOW VIRTUALLY UNHEARD
OF.
THE TRANSITION HAS BEEN ABRUPT
AND VERY RAPID AND PROBABLY FELT
IN YOUR FIELDS AS WELL.
NEXT SLIDE.
SO WHERE DO WE GO NEXT IN
BUILDING ON THE MOMENTUM OF THE
CANCER MOONSHOT?
SO WE HAVE A NUMBER OF PRIORITY
AREAS THAT I WANT TO JUST LET
YOU KNOW ABOUT THAT WE ARE
TACKLING WITH SOME OF THE SAME
MINDSET THAT CAME FROM THIS
IGNITION THAT CAME FROM THE
CANCER MOONSHOT.
AS YOU'VE SEEN IN THE NEWS,
THERE IS A VERY MARKED AND
DISTINCT RISE IN CANCERS THAT
ARE MORE COMMONLY IN OLDER AGE
GROUPS HAPPENING TO PEOPLE IN
THEIR 20s, 30s, AND 40s.
COMMON CANCERS COLORECTAL,
ENDOMETRIAL, KIDNEY CANCER, WE
ARE SEEING A 79% INCREASE IN
THESE CANCERS, INCREASE THAT IS
NOT COMPLETELY UNIFORM AND AN
AREA WE NEED TO STUDY.
WE ARE BRINGING THE SAME KIND OF
APPROACH THAT WE BROUGHT TO THE
OVERALL CANCER MOONSHOT TO UNDER
THE IDEOLOGY, THE BIOLOGY, THE
BEST TREATMENT AND HOW BEST TO
CARE FOR PATIENTS WHEN LIFE
AFTER CANCER WITH WHEN THAT
CANCER IS DIAGNOSED IN YOUNG
EARLY ADULTHOOD.
WE ALSO ARE TAKING THIS
MENTALITY TO MODERNIZING OUR
CLINICAL STUDIES.
JUST ONE EXAMPLE IS MYELOMATCH.
DURING THE CANCER MOONSHOT WAS
OUR FIRST MATCH STUDY, WHICH
TESTED MATCHING GENETIC PROFILE
OF THE PATIENT TO A DRUG.
TODAY WE HAVE, AS YOU HAVE SEEN,
A PLETHORA OF MORE AVAILABLE
DRUGS AND MORE KNOWLEDGE ABOUT
WHAT THOSE GENETIC MUTATIONS
MEAN.
SO THE NEXT GENERATIONS ARE
PROGRAMS LIKE MYELOMATCH TAKE
PATIENTS WITH ACUTE MYELOID
LEUKEMIA AND MATCH THEM OVER THE
COURSE OF THEIR DISEASE TO THE
BEST TREATMENT BASED ON THEIR
PROFILE.
THIS TRIAL HAS ALREADY LAUNCHED
AND AS SCREENED SEVERAL HUNDRED
PATIENTS IN THE FIRST COUPLE OF
MONTHS IT HAS BEEN AVAILABLE AND
WILL BE A VERY EXCITING NEW WAY
OF THINKING ABOUT HOW WE TAKE
CARE OF HERKS EMATOLOGICAL
MALIGNANCIES.
ONE OF THE BURDENS WE LEARNED
ABOUT THE CANCER MOONSHOT
ASSESSMENTS WERE HOW HARD IT IS
TO DO A CLINICAL TRIAL BECAUSE
OF THE MANY REQUIREMENTS FOR
REQUIRING PATIENT ELIGIBILITY
AND TESTING, SOME OF WHICH MIGHT
NOT MAKE COMMON SENSE.
SO PRAGMATIC IS TAKING TRIALS
AND REMOVING THOSE BARRIERS TO
ACCESS.
WHAT DO YOU REALLY NEED TO BE
ABLE TO TEST THIS?
THIS REQUIRED CLOSE
COLLABORATION WITH THE FDA AND
WORKING WITH DRUG COMPANIES AND
INVESTIGATORS AND PATIENTS TO BE
ABLE TO PUT TOGETHER A VERY
HIGHLY STREAMLINED TRIAL.
THIS IS A RANDOMIZED PHASE 3
CLINICAL TRIAL TESTING FIRSTLINE
THERAPY THAT IN ONE YEAR HAS
ALMOST COMPLETELY ENROLLED AND
WILL BE DONE SOON.
THIS IS WHERE WE NEED TO GO FOR
MANY STUDIES.
WE CAN'T WAIT YEARS FOR THE
INFORMATION WE NEED AND WE NEED
TO MAKE TRIALS MORE ACCESSIBLE
WITHOUT BEING OVERLY BURDENSOME.
I WANT TO POINT TO THE WAY WE
CAN USE AI AND OTHER DIGITAL
TOOLS TO MODERNIZE CLINICAL
STUDIES.
WE JUST PUBLISHED RECENTLY ON A
PILOT STUDY TO USE TRIAL GPT.
LIKE CHATGPT TO TRY TO MATCH THE
PATIENT AND THEIR CONDITION WITH
THEIR SET OF CIRCUMSTANCES TO
THE RIGHT TRIAL SO THAT WE DON'T
HAVE TO SPEND UNNECESSARY TIME
GETTING TO THE RIGHT PLACE.
I ALREADY MENTIONED IN THE
PRAGMATIC TRIAL PARTNERING WITH
THE FDA IN THE CLINICAL TRIAL
SPACE.
THE CLINICAL TRIAL INNOVATION
UNIT WAS STARTED WITH DR.
BERTAGNOLLI WHEN SHE WAS THE NCI
DIRECTOR TO TEST NOVEL DESIGNS,
BIOMARKERS OR STRATEGIES THAT
ALLOW FOR MORE EFFECTIVE CARE
AND NEW DATA METHODOLOGIES.
ROAD TEST THEM AT THE FDA WITH
THE CLINICAL TRIAL NETWORKS AND
NCI STAFF BEFORE WE BRING THEM
FORWARD.
WE ARE ALSO VERY INVESTED IN HOW
WE BRING CLINICAL RESEARCH INTO
THE COMMUNITY.
80% OF CANCER PATIENTS RECEIVE
THEIR CARE OUTSIDE OF ACADEMIC
CENTERS AND WE KNOW THERE IS NO
CANCER FOR WHICH WE HAVE ALL OF
THE ANSWERS.
THERE IS A GREAT NEED FOR
CLINICAL RESEARCH TO BE
AVAILABLE FOR PATIENTS ACROSS
THE SPACE.
SO WE CHARGED A WORKING GROUP
EARLIER THIS YEAR WHICH JUST
REPORTED OUT EARLIER IN DECEMBER
ASKING THEM HOW TO CRAFT NEW
APPROACHES TO BRING LEADING EDGE
CLINICAL RESEARCH TO CLINICAL
SETTINGS.
IT WAS CHAIRED BY BILL DAHUT AND
RAY OSAROGIAGBON FROM TENNESSEE
WHO IS A N CORE LEADER.
A LEADER OF A NATIONAL ONCOLOGY
RESEARCH PROGRAM.
LIVING AND WORKING IN COMMUNITY
CLINICAL ONCOLOGY.
WE PARTNERED ACROSS FEDERAL
AGENCIESs AS YOU HEARD ABOUT
WITH THE CANCER CABINET, VA,
CMS, FDA, AND ACROSS ADVOCACY
GROUPS AS WELL TO ASK THEM TO
BRING SIMILAR TYPE OF
RECOMMENDATIONS WE SAW WITH THE
BLUE RIBBON PANEL, FOR EXAMPLE.
THEY BROUGHT US TWO MAJOR
RECOMMENDATIONS.
THE FIRST IS TO BREAK DOWN THE
SEPARATION OF CLINICAL RESEARCH
AND CLINICAL CARE AND BEGIN TO
THINK ABOUT THIS IN A MUCH MORE
OF A CONTINUITY.
GREAT EXAMPLES WITHIN THERE THAT
WE'LL BE TACKLING OVER THE NEXT
YEARS.
HOW DO WE GET TO ERADICATING
CERVICAL CANCER.
BRINGING ADDITIONAL SCREENING
FOR PROSTATE CANCER INTO
COMMUNITIES AS WELL AS HOW WE
BRING CLINICAL RESEARCH INTO
COMMUNITIES.
SO WE ARE REALLY EXCITED TO WORK
THROUGH THAT LIST.
THE SECOND WAS A PROPOSAL TO
EXPAND CLINICAL RESEARCH SITES
ACROSS COMMUNITY NETWORKS AND
PARTNERING WITH PHYSICIANS AND
PROVIDERS ACROSS THE COUNTRY.
SO A REALLY FORWARD THINKING AND
EXCITING SET OF RECOMMENDATIONS
THAT CAME FROM THIS GROUP.
AND SO I'M GETTING TO THE END.
WHAT I THINK THIS HIGHLIGHTS,
WHAT CAME FROM THE MOONSHOT WAS
NOT JUST A BUNCH OF GRANTS AND A
BUNCH OF PAPERS, IT WAS A
MINDSET THAT DEFINES A DIFFERENT
ERA OF BIOMEDICAL RESEARCH.
WE ALL KNOW AN ERA WHERE
EVERYTHING WAS TOP DOWN BY
NECESSITY.
DOCTOR KNOWS BEST.
AN ERA WE LIVED IN BEFORE,
RECOGNIZING THE COLLABORATION OF
MULTIDISCIPLINE CARE.
NOW BECAUSE OF THE MOONSHOT AND
THE WORK TO BRING MORE COMMUNITY
ENGAGEMENT, TO BRING MORE
ENGAGEMENT WITH EARLY STAGE
INVESTORS, WE HAVE AN ERA WE ARE
CALLING 360 WHICH CLOSES THE
LOOP IN FULL CIRCLE AROUND THE
RESEARCH, THE CARE AND THE
PATIENT.
SO THAT WE CAN DELIVER THE BEST
IN WHAT WE DO.
AND SO THIS JUST HIGHLIGHTS HOW
MANY OF THE DIFFERENT THINGS I
HAVE TALKED ABOUT HERE TODAY ARE
HUMAN TISSUE ATLAS NETWORK
CONNECTS TO EVEN THE WORK OF THE
HPV TESTING.
SO IT ALL FITS TOGETHER AND
BRINGS THE PATIENT INTO THE MIX
AS WELL.
NCI INNOVATION WORKING IN 360
IS, I THINK, THE ULTIMATE OUTPUT
OF THE CANCER MOONSHOT.
THAT IS MY LAST SLIDE AND I'LL
TAKE THE NEXT AND THANK YOU VERY
MUCH.
I'D BE HAPPY TO TAKE QUESTIONS.
>> OKAY.
THANK YOU.
COULD WE PULL UP -- YEAH.
# THANKS.
SO WHO WOULD LIKE TO BEGIN THE
QUESTIONS?
YES.
GISELLE.
>> FIRST OF ALL, GOOD MORNING,
EVERYONE.
I APOLOGIZE FOR MY CONTINUED
ZOOM CHALLENGES.
I'M ACTUALLY ON MY PHONE NOW
TRYING TO CONNECT AND RESTARTING
MY COMPUTER.
FIRST OF ALL, I JUST WANT TO
THANK YOU FOR THIS PRESENTATION.
HOW INSPIRING IS IT TO HEAR WHEN
WE PUT OUR EFFORTS AND ATTENTION
TO A PARTICULAR PROBLEM WHAT WE
ARE ABLE TO ACCOMPLISH.
I HAVE TWO QUESTIONS FOR YOU.
THE FIRST IS ONE OF THE THINGS I
WAS LISTENING FOR AND MAY HAVE
MISSED ATTENTION TO FINANCIAL
TOXICITY AROUND CANCER TREATMENT
AND CARE.
AND WONDERING -- I CAN SEE
PLENTY OF PLACES WHERE IT WOULD
FIT IN, BUT WONDERING IF THERE
IS ANY ATTENTION TO THAT AS IT
CONTRIBUTES TO SUFFERING FOR
CANCER SURVIVORS.
AND SECOND OF ALL, GIVEN WHAT
YOU HAVE BEEN ABLE TO
ACCOMPLISH, THERE ARE SO MANY
OTHER CONDITIONS THAT COULD
BENEFIT FROM THIS KIND OF
INTENTIONAL APPROACH,
COLLABORATION AND SYNERGY.
WHAT ARE THE LESSONS LEARNED?
YOU SUGGESTED SEVERAL DURING THE
COURSE OF YOUR PRESENTATION?
I WONDER IF YOU CAN DISTILL THAT
DOWN AS WE THINK ABOUT OTHER
CONDITIONS THAT NEED THIS KIND
OF ATTENTION.
FOR EXAMPLE MENTAL HEALTH IN
YOUTH AND THERE ARE A WHOLE
RANGE OF THINGS WE COULD THINK
ABOUT.
>> ABSOLUTELY.
I WILL TAKE FINANCIAL TOXICITY
FIRST.
IT IS A LITTLE MORE RECENT THAT
WE HAVE BEEN ABLE TO PUT A NAME
TO THAT AND SO WE'RE REALLY
STARTING TO INCORPORATE THAT IN
THE WORK THAT WE DO.
FOR EXAMPLE, IN ASKING PATIENTS
MORE DELIBERATELY HOW THE
FINANCIAL EFFECTS OF CANCER
TREATMENT ARE IMPACTING THEIR
JOB, THEIR LIVELIHOOD OR
DECISIONS THAT THEY'RE MAKING.
SO WE'VE BEEN FUNDING RESEARCH
IN THIS AREA FOR QUITE SOME
TIME.
IN OUR ANNUAL CANCER PLAN WE
HIGHLIGHTED FOUR AREAS OF FOCUS
AND THAT WAS ONE OF OUR AREAS
THAT WE SAID THAT NCI REALLY HAS
TO STEP INTO IS ADDRESSING
FINANCIAL TOXICITY.
STUDYING WAYS TO BETTER APPROACH
THIS.
TO YOUR SECOND, I THINK THE
BIGGEST LESSON LEARNED IS THAT
IT IS IMPORTANT TO HAVE THE
VOICES OF MANY MORE STAKEHOLDERS
TO BE ABLE TO MAKE REALLY GOOD
INFORMED RESEARCH PRIORITIES AND
DECISIONS.
WE ARE DEFINITELY APPLIED TO
THAT EARLY ONSET CANCER.
WE ARE IN THE EARLY PHASE OF
DOING FOCUS WORK AND TALKING TO
PEOPLE EXPERIENCING EARLY ONSET
CANCERS.
AND LEARNING A LOT ABOUT WHERE
SOME OF THE GAPS ARE.
AND IT CAN ALLOW US TO PUT SOME
OF OUR RESEARCH DOLLARS TO
PLACES THAT WILL HAVE REAL
IMPACT IN A MUCH MORE MEANINGFUL
WAY VERY QUICKLY.
I THINK THE BIGGEST LESSON
LEARNED THOUGH IS HAVING A
MULTIPRONGED APPROACH THAT WE
KEEP COMING BACK TO AND BEING
ABLE TO KEEP TESTING EACH OF
THOSE 10 RECOMMENDATIONS AND
MAKING SURE WE KEEP MAKING
ADVANCES ON THEM.
I REMEMBER WATCHING THIS IN ITS
EARLY DAYS AND IT WOULD HAVE
BEEN EASY TO FOCUS ON ONE AND
LET ANOTHER SLIDE, BUT BECAUSE
THEY WERE ALL THERE AND ALL
BEING REPORTED ON AND BEING
EVALUATED, IT KEPT EACH ONE
MOVING.
AND YOU SAW IN THERE THAT THEY
OVERLAPPED ACCEPTING A SPACE FOR
OVERLAP AND THAT COULD MAKE THE
FOCUS ON PEDIATRIC CANCERS AND
IMMUNOTHERAPY NETWORKS, ALLOWING
THOSE TO COME TOGETHER.
WE ARE KILLING TWO BIRDS WITH
ONE STONE IN THAT PARTICULAR
CASE.
IT ALSO MADE MORE ADVANCES FOR
BOTH THE IMMUNOLOGISTS AND
PEDIATRIC ONCOLOGY SPACE.
>> THANK YOU.
GARTH.
>> MY QUESTION IS SPECIFIC TO
THE CHILD GPT PAPER.
IF YOU COULD CLICK INTO THE
IMPACT DATA OR LESSONS LEARNED
AND WHETHER OR NOT TOOLS LIKE
LARGE MODELS IN THAT CONTEXT
WERE ACTUALLY BENEFICIAL.
>> YEAH.
SO IT WAS A FIRST PHASE PILOT.
THERE IS A SECOND PHASE PILOT
BECAUSE WE LEARNED THINGS ABOUT
PLACES WHERE IT WASN'T ALWAYS
RIGHT AND PLACES WHERE IT COULD
CREATE CHALLENGES, BUT THE
OVERALL IMPRESSION WAS THAT THIS
IS GOING TO BE A REAL GAME
CHANGER.
I MEAN, IF YOU JUST THINK ABOUT,
I WAS TALKING TO A PATIENT JUST
A COUPLE OF WEEKS AGO WHO
REACHED OUT, WHO HAS MEANS, WHO
WANTS TO TRAVEL TO FIND THE
RIGHT CLINICAL TRIAL FOR THEIR
DISEASE.
ASKING ME SHOULD THEY GO DANA
FA
FARBER OR DUKE OR UCLA.
TODAY IT INVOLVES A LOT OF PHONE
CALLS, RELYING ON EXPERTS AND
TIME YOU SPENT IN PLANES, CARS
AND HOTEL ROOMS, WHICH IS REALLY
NOT RIGHT.
AS WE LEARN TO USE TOOLS LIKE
CHATGPT IN OUR EVERY DAY
PLANNING AND DISCOVERY, THIS
WILL BE A REAL OPPORTUNITY.
IT JUST HAS TO BE DONE RIGHT.
PATIENT CARE IS SAFETY FIRST AND
MAKING SURE WE DON'T INCORPORATE
ANYTHING THAT IS INCORRECT OR
LEADS PEOPLE ASTRAY.
SO THE NEXT PHASE IS GOING TO
TAKE IT TO THE NEXT LEVEL AND
REALLY ROAD TEST SOME OF THE
PARTICULAR CHALLENGES.
#
>> THANK YOU.
RUSS, YOU ARE NEXT, PLEASE.
YOU ARE ON MUTE.
>> CAN YOU HEAR ME NOW?
>> YES, WE CAN.
>> OKAY.
APOLOGIES.
MY CONGRATULATIONS AS WELL DR.
RATHMELL FOR LOOKING AT THE
OVERALL CANCER STATISTICS.
IT IS DRAMATIC HOW MUCH IMPACT
ALL OF THIS RESEARCH HAS HAD ON
OUR NATIONAL BURDEN OF DISEASE
AND SURVIVAL.
THE TWO QUESTIONS I HAVE FOR YOU
ARE NUMBER ONE, IN A BROADER
SENSE IN TERMS OF THE FINANCIAL
TOXICITY ISSUE, MANY OF THESE
GREAT THERAPIES COMING OUT OF
THESE STUDIES ARE REMARKABLY
EXPENSIVE, LIKE CAR T IS HALF A
MILLION TO A MILLION DOLLARS PER
PATIENT.
I DON'T THINK THAT IS A
SUPPORTABLE COST IN OUR HEALTH
ECOSYSTEM WHERE I'M NOT ALLOWED
TO GET AN MRI FOR SOMEONE WITH
CHRONIC BACK PROBLEMS OVER COST
ISSUES.
IS THERE A THOUGHT TO
PRIORITIZING SECOND-LEVEL
APPROACHES THAT WOULD HAVE
HIGHER COST EFFECTIVENESS?
I'M THINKING OF OFF THE SHELF
CAR T?
HOW IS THAT PRIORITIZED IN THE
NCI PORTFOLIO AND THE SECOND AND
RELATED QUESTION, ANY TIME THERE
IS A BOLUS FUNDING, WHEN IT ENDS
THERE ARE SOME REAL CHALLENGES.
YOU'VE LAUNCHED A CAREER OF
ABOUT 80 YOUNG INVESTIGATORS
WITH THE MOONSHOT MONEY, WHICH
IS FANTASTIC.
YOU HAVE SUPPORTED SOME
INCREDIBLE WORK IN AT LEAST 20
DIFFERENT DIRECTIONS.
THAT EYE CHART GRAPH SHOWING
JUST SOME OF THE WORK HERE IS
REMARKABLE.
BUT IT IS NOT SUSTAINABLE
WITHOUT THAT CONTINUED LEVEL OF
FUNDING AND IF YOU COULD SPEAK
TO SOME OF THE CHALLENGES YOU
ARE HAVING AS AN INSTITUTE
DIRECTOR NOW IN THE POST
MOONSHOT TRANSITION.
>> YEAH.
I'LL TAKE FIRST THE CAR T.
SO THIS IS A FIELD IN EVOLUTION
AND SOME OF THE WORK WILL BE
VERY CUSTOMIZED AND EXPENSIVE
BEFORE IT BECOMES OFF THE SHELF.
THAT IS A REAL AREA FOR FOCUS
AND IN FACT THERE ARE STUDIES
TRYING TO MOVE INTO THAT
DIRECTION AS WELL AS COMPANIES
THAT ARE TAKING THINGS INTO THAT
DIRECTION.
I THINK BACK TO, I WAS A POST
DOCTORAL FELLOW AT THE
UNIVERSITY OF PENNSYLVANIA WHEN
KARL JUNG WAS PIONEERING CAR Ts
AND NO ONE THOUGHT IT COULD BE
DONE FOR ANY MORE THAN ONE OR
TWO PATIENTS AT A TIME AND HERE
WE ARE TODAY.
SO THE OPPORTUNITIES ARE REAL.
WE ALSO NEED ECONOMIC REALITIES
TO HELP SHAPE THEM TO THE PLACE
WHERE THEY NEED TO BE.
IN TERMS OF THE EXPLOSION OF
OPPORTUNITY THERE, JUST ONE
EXAMPLE OF THE WAYNICK CAN HELP
WITH THAT IS WE DO GMP
PRODUCTION AT THE FREDERICK
NATIONAL LABS AND SO SOME OF
THESE STUDIES THAT COULD
PREVIOUSLY ONLY BE DONE AT ONE
INSTITUTION WE CAN HELP TO
DERISK AND ALLOW THE SAMPLES TO
BE PAIR AND DISTRIBUTED SO THEY
CAN BE OPEN IN MULTISITE TRIALS
AND ADVANCE THIS AND GET TO THE
PLACE WHERE WE ALL WANT TO BE
WHERE THIS IS GIVEN TO THE RIGHT
PATIENT AT THE RIGHT TIME AND AT
THE RIGHT COST.
YOU ARE EXACTLY RIGHT TO POINT
OUT THAT THE END OF THE MOONSHOT
FUNDING WAS NOTICEABLE.
OUR DELTA IN REAL DOLLARS WAS
$96 MILLION DECREASED THIS YEAR,
EVEN THOUGH WE DID SEE AN
INCREASE TO OUR BASE.
YES.
THAT DOES MEAN A CHALLENGE FOR
NEW INVESTIGATORS AND A MAJOR
EFFORT WE ARE UNDERTAKING TO
REPRIORITIZE TO CONTINUE TO
SUPPORT OUR INVESTIGATORS ACROSS
THE NATION.
WE DO SEE EXACTLY THE PHENOMENON
YOU DESCRIBED.
THE NUMBER OF INVESTIGATORS
PUTTING IN GRANTS IS HIGHER
TODAY AND THAT'S ACTUALLY BEEN
THE BIGGEST BARRIER TO US BEING
ABLE TO CHANGE OUR PAY LINE AND
GET IT UP MORE WHEN WE HAVE A
BIGGER DENOMINATOR OF GRANTS
COMING IN THE DOOR NOT TO
MENTION THE GRANTS ARE GETTING
BIGGER.
THESE ARE THE ECONOMIC ARGUMENTS
I'M USING WHEN TALKING TO
MEMBERS OF CONGRESS TO
UNDERSTAND THE TREMENDOUS NEED
FOR THE WORK, THE RETURN ON
INVESTMENT AND THE OPPORTUNITIES
IT PROVIDES IN TERMS OF A
SKILLED WORKFORCE IN BRINGING
NEW TREATMENTS AND THEY WORRY
ABOUT THE EXPENSE OF THE
TREATMENTS, BUT NEW
OPPORTUNITIES FOR PREVENTION,
EARLY DETECTION, TO MANAGE
CANCERS BEFORE THEY ARE HIGHLY
ADVANCED AND FOCUSES AS WELL ON
SURVIVAL AND SURVIVORSHIP.
SO THIS IS WORK THAT COSTS REAL
MONEY IF WE CONTINUE TO BE HELD
TO A VERY TIGHT BUDGET, IT WILL
FEEL THE SQUEEZE ACROSS THE
COUNTRY.
>> THANK YOU.
MONICA, BACK TO YOU.
WE ARE JUST ABOUT AT TIME.
>> OKAY.
FANTASTIC.
I JUST WANT TO EMPHASIZE JUST
ONE OTHER, I THINK FOR ME,
INCREDIBLE COMPONENT OF THE
MANAGEMENT THAT NCI HAS
ACCOMPLISHED UNDER DR.
RATHMELL'S LEADERSHIP AND THAT
IS JUST LOOK AT THE TREMENDOUS
IMPACT OF BOTH INVESTMENTS IN
INFRASTRUCTURE, SUSTAINED
INFRASTRUCTURE THAT MANY PEOPLE
CAN TAKE ADVANTAGE OF AS WELL AS
THE PARTNERSHIPS THAT ARE
CREATED BY PUTTING FUNDING INTO
COLLABORATIVE ACTIVITIES.
I THINK THAT ALSO SOMETHING WE
RECOGNIZE AS A TREMENDOUS
ACCELERANT.
THANK YOU FOR A WONDERFUL
PRESENTATION AND, BOY, WHAT AN
ACTION-PACKED YEAR YOU'VE HAD.
CONGRATULATIONS.
>> THANK YOU.
>> SO MUCH ACCOMPLISHED IN SUCH
A SHORT PERIOD OF TIME.
WE ARE GOING TO MOVE FORWARD TO
OUR NEXT PRESENTATION.
WE'RE GOING TO HEAR FROM DR.
NORA VOLKOW.
AND SHE IS GOING TO TALK TO US
ABOUT THE ABCD STUDY.
OUR STUDY OF ADOLESCENT BRAIN
COGNITIVE DEVELOPMENT.
NORA, TAKE IT AWAY.
>> MONICA, THANKS A LOT.
AND GOOD MORNING, EVERYONE.
IT IS A PLEASURE TO BE ABLE TO
SPEAK TO YOU ABOUT THE
ADOLESCENT BRAIN COGNITIVE
DEVELOPMENT STUDY.
I JUST WISH I WERE THERE WITH
ALL OF YOU.
WE ENDED UP VIRTUAL.
SO THE ABCD STUDY IS ACTUALLY
REMARKABLE IN MANY WAYS.
I WILL START BY SAYING IT IS
EXEMPLIFIES SOMETHING THAT
ACTUALLY HIGHLIGHTS THE ABILITY
OF THE INSTITUTES TO COME
TOGETHER AND TO FORM A
FUNCTIONAL INTEGRATION EFFORT TO
ADVANCE THE SCIENCE IN WAYS THAT
IT CAN HELP EVERYONE.
THE IDEA STARTED I THINK ONE
DECADE AGO, PROBABLY MORE, AND
IT WAS BOTH ME AND DR. -- AND
THE NATIONAL INSTITUTES OF
ALCOHOL ABUSE AND ALCOHOLISM
DISCUSSING HOW VALUABLE IT WOULD
BE FOR US IN THE FIELD OF
ADDICTION TO UNDERSTAND WHAT IS
IT ABOUT THE HUMAN BRAIN DURING
ADOLESCENTS THAT MAKES IT SO
VERY VULNERABLE TO THE USE OF
DRUGS, TO PEER PRESSURE?
AND ULTIMATELY PUTS THEM AT SUCH
A HIGH RISK OF ADDICTION AS WELL
AS OTHER BEHAVIORS THAT ARE ONE
OF THE MAIN CAUSES OF MORBIDITY
AND MORTALITY AT THIS AGE.
WE WERE SALIVATING BECAUSE WE
WERE AWARE OF ALL OF THE
TECHNOLOGIES BEING DEVELOPED
THAT ALLOW YOU LOOK INTO THE
HUMAN BRAIN NONINVASIVELY.
WE PUT OUR HEADS TOGETHER AND
THE ABCD WAS BORN.
HOW ABOUT IF WE DO IT JOINTLY.
WE DID IT JOINTLY WITH THE
NATIONAL CANCER INSTITUTE, TO
PUT RESOURCES TO DO THE LARGEST
STUDY EVER DONE AND MONITOR THE
DEVELOPMENT OF TRAJECTORIES OF
THE HUMAN BRAIN THEREFORE IN A
PERSPECTIVE WAY ACROSS A LARGE
CORPORATE STUDY.
THIS IS HOW IT WAS BORN.
THE FIRST WE PROVIDED THE
FUNDING AND THE FIRST
RECRUITMENT OF CURE IN 2016 AND
WE WERE NOW 18 YEARS LATER.
BUT WHAT ACTUALLY DROVE US TO
COME TO THE IMPORTANCE OF
LAUNCHING THIS ABCD APART FROM
RECOGNIZING HOW VALUABLE IT WAS?
THERE WERE MANY ISSUES THAT WERE
HAPPENING AT THAT TIME THAT LED
TO IT.
IF I CAN HAVE THE NEXT SLIDE.
NUMBER ONE, THE RECOGNITION ALL
ALONG IN MEDICINE THAT
ADOLESCENTS IS NOT JUST A PERIOD
OF HIGH RISK FOR TAKING DRUGS,
BUT IF YOU LOOK AT TWO
CHIMPBT -- WHO HAS BEEN LEADING
THE STUDY WHO WERE AT THE TIME
OF 20169 TO 10 YEARS OLD SHLGS
WHICH WERE THE CHILDREN WE WERE
RECRUITING FOR ABCD.
FOLLOWED UP FOR SIX YEARS.
WHAT IS STRIKING IS HOW
DRAMATICALLY THESE CHILDREN NOW
TURNING DOSH ARE CHANGING AND
THIS CONNOTES THE IMPORTANCE OF
UNDERSTANDING BETTER WHAT ARE
THE BIOLOGICAL UNDERPINNINGS AND
THE INFLUENCE OF THOSE
BIOLOGICAL INFLUENCES BY
ENVIRONMENTAL FACTORS.
SO THE RECOGNITION THAT THIS IS
A PERIOD OF TREMENDOUS
TRANSITION AND CHANGE.
AT THAT TIME ALSO WE WERE VERY
SENSITIVE AND THIS WAS AN
IMPORTANT QUESTION IN OUR
COUNTRY THAT THERE WERE
SIGNIFICANT CHANGES IN THE
PATTERNS OF DRUG USE AMONG
TEENAGERS AND THE LANDSCAPE WAS
CHANGING QUITE DRAMATICALLY.
THE PARTICULAR CONCERN WAS
LEGALIZATION OF MARIJUANA BY
MULTIPLE STATES.
IT IS STILL AN ILLICIT DRUG,
STATES WERE MAKING IT LEGAL FOR
RECREATION OR MEDICINAL
PURPOSES.
THE USE OF SUBSTANCES IN
ADOLESCENT CREATES MORE
CONSEQUENCES THAN LATER IN THE
LIFE.
THE BRAIN IS TREMENDOUSLY
NEUROPLASTIC AND AS A RESULT OF
THAT DRUGS ARE MORE LIKELY TO
PRODUCE CHANGES THAT ARE GOING
TO BE LONG LASTING.
INDEED, THE EPIDEMIOLOGICAL AND
DATA DOES CORROBORATE THIS TO BE
THE STATE.
THE LEGALIZATION WOULD MAKE
TEENAGERS MORE LIKELY TO TAKE
DRUGS AND WE WANTED TO
UNDERSTAND THE CONSEQUENCES.
EVEN THOUGH THERE HAS BEEN A LOT
OF WORK ON UNDERSTANDING HOW THE
USE OF WORKS IN ADOLESCENTS
AFFECTS THE BRAINS, THERE HAVE
BEEN A LOT OF QUESTIONS REGARDS
THESE ASSOCIATIONS BROUGHT UP BY
EPIDEMIOLOGICAL STUDIES THAT
SHOW THAT CHILDREN THAT GET
EXPOSED TO MARIJUANA ARE MUCH
MORE LIKELY TO END UP WITH
PSYCHOTIC DISEASES INCLUDING
SCHIZOPHRENIA AND DROPOUT OF
SCHOOL AND PERFORM WORSE LATER
ON COGNITIVE TESTS.
THE CHALLENGE WAS HOW DO YOU
KNOW THESE KIDS WERE NO
DIFFERENT TO START WITH AND THAT
IS WHY THEY CONSUME MARIJUANA?
IT WAS A VERY IMPORTANT QUESTIN
TO ASK AND NOT JUST FOR
MARIJUANA.
IT WAS ALSO IMPORTANT TO ASK FOR
ALCOHOL, FOR NICOTINE.
IMPORTANTLY WE RECOGNIZE THERE
HAS BEEN A SHIFT IN THE PATTERNS
OF DRUG USE BY TEENAGERS.
FOR EXAMPLE, AT THAT TIME THERE
WAS ELECTRIC CIGARETTES.
CLOSE TO 30% OF TEENAGERS WERE
ALREADY USING WITHIN TWO YEARS
THESE VAPING DEVICES,
OUTSTANDING TOOLS TO DELIVER
HIGH DOSES OF DRUGS AND VERY,
VERY FAST.
SO WE NEEDED TO KNOW THAT.
MEANWHILE THE MOMENT WAS ALSO
RIGHT BECAUSE THE TECHNOLOGY
ADVANCED IN SUCH A WAY THAT MANY
OF THE ACADEMIC CENTERS HAD
ACCESS TO SOPHISTICATED IMAGING
DEVICES THAT ALLOWED IN DEPTH
CHARACTERIZATION OF THE BRAIN.
NONINVASIVELY, THE ACCESS TO
COMPUTERS AND WEARABLE DEVICES
PROVIDE AN IN DEPTH
CHARACTERIZATION AND THE
REPOSITORIES THAT ARE COLLECTING
DATA ACROSS DIFFERENT
INSTITUTIONS ALLOWED THEM THE
POSSIBILITY OF GENERATING A
LARGE COHORT STUDY THAT WOULD BE
EVALUATED IN GREAT DETAIL
MULTIPLE PARAMETERS RELEVANT TO
HUMAN BRAIN DEVELOPMENT.
THERE WAS AN ENORMOUS INTEREST
FROM OTHER INSTITUTES THAT
IMMEDIATELY JUMP IN.
FOR THE NATIONAL INSTITUTES OF
MENTAL HEALTH IT WAS
EXTRAORDINARILY RELEVANT.
THE MAJORITY OF THE SYMPTOMS OF
PSYCHIATRIC DISEASES OCCUR
DURING ADOLESCENTS AND THERE HAS
ALWAYS BEEN THAT RECOGNITION OF
HOW IMPORTANT IS BRAIN
DEVELOPMENT TO MENTAL ILLNESS
AND, IN FACT, DESCRIBE MENTAL
ILLNESS AND ADDICTION AS
DEVELOPMENTAL DISORDERS.
SEVEN INSTITUTES JUMP IN AND
FUND THE STUDY AND THAT IS HOW
IT WAS BORN.
THE RESEARCH OBJECTIVES WERE
SIMPLE.
YOU GO TO THE PEDIATRICIAN THAT
MEASURES THE HEIGHT AND WEIGHT
OF THE CHILD AND THERE ARE
SCALES TO TELL YOU THAT HEIGHT
OR WAIT ARE NORMAL OR OUTSIDE OF
NORMATIVE STANDARDS.
NOW WE HAVE TOOLS THAT ALLOW US
TO LOOK AT THE BRAIN DEVELOPMENT
WITHIN THE STANDARD.
THIS WAS NOT TRIVIAL BECAUSE IN
THE FIELD OF PSYCHIATRY, THE
CHILD STARTS TO FAIL AT SCHOOL,
HAVE PROBLEMS WITH THEIR
FRIENDS.
THEY ISOLATE.
YOU TAKE THEM TO THE
PEDIATRICIAN OR THE PSYCHIATRIST
THIS IS PART OF ADOLESCENCE.
LET'S FOLLOW UP FOR SIX MONTHS.
WOULDN'T IT BE IMPORTANT AND
VALUABLE TO HAVE A MEASURE WHERE
IF YOU START TO HAVE SYMPTOMS
THAT ARE DISSTRESSING TO
EVALUATE WHETHER THE BRAIN IS
FOLLOWING THE TRAJECTORY AND
STANDARD THAT GIVE YOU A SENSE
OF SOMETHING THAT MAY BE
DEVELOPING.
WE WANTED TO DEVELOP A LARGE
ENOUGH STUDY THAT WOULD ALLOW US
FOR THE FIRST TIME START TO
GENERATE THOSE STANDARDS OF WHAT
THE HUMAN BRAIN SHOULD LOOK
LIKE, UNDERSTANDING THE
TREMENDOUS DIVERSITY.
SO AND THAT RELATED ALSO IN
PARALLEL TO COGNITIVE PROCESSES.
WE WERE ALSO AT THAT STAGE VERY
MUCH, I MEAN AN AREA OF
TREMENDOUS INTEREST, OF COURSE,
HAS BEEN HOW DO GENES EFFECT
DISEASE?
HOW DO GENES EFFECT DISEASE?
IN THE BRAIN A NEUROPLASTIC
ORGAN, BY THE NATURE OF THIS
SYSTEM, THE BRAIN DEVELOPS FOR
20 YEARS BECAUSE YOU NEED TO
ACTUALLY ENCOUNTER THE
ENVIRONMENT IN ORDER TO CREATE
ITS ARCHITECTURE AND ITS
CONNECTIONS.
SO WE WANTED TO SEE HOW GENES
AND ENVIRONMENT INFLUENCE THEM.
WE WANTED TO BE ABLE TO MONITOR
HOW BEHAVIORS THAT WE HAVE SOME
GUIDELINES FOR ULTIMATELY AFFECT
THEM SO WE CAN HAVE EVIDENCE TO
SUPPORT THOSE GUIDELINES.
FOR EXAMPLE, PHYSICAL
ACTIVITIES, SLEEP, SCREEN TIME,
THE EFFECT OF OTHER EXPERIENCES
IN TERMS OF EDUCATION.
HOW DO THEY INFLUENCE BRAIN
DEVELOPMENT?
IMPORTANTLY, MENTAL ILLNESS IS
ONE OF THE MAIN FACTORS OF
BURDEN OF DISEASE AMONG THE
YOUNG.
AND SO WE BELIEVE THAT WAS VERY
CRUCIAL TO PRIORITIZE.
MENTAL ILLNESS CO-MINGLES WITH
SUBSTANCE USE.
THERE IS BIDIRECTIONALITY OF
THOSE TWO AND CREATES
TREMENDOUS, TREMENDOUS HARM.
AS I SAY, THAT WHOLE LANDSCAPE
OF SUBSTANCES WERE RAPIDLY
CHANGING AND WE WANTED TO
UNDERSTAND HOW THAT INFLUENCED
BRAIN TRAJECTORIES.
HOW DID WE ACHIEVE THIS?
WELL, LIKE ANYTHING ELSE AT THE
NIH, WE RELIED ON RESEARCHERS
ACROSS ALL OF THE COUNTRY.
WE REQUESTED PROPOSALS THAT
BASICALLY WITH THE GOAL OF DOING
BRAIN STUDY FOR 12 YEARS.
RECRUITMENT OF CHILDREN 9 TO 10.
NOT BECAUSE WE DIDN'T THINK
INFANCY IS IMPORTANT, IT IS
CRUCIAL, BUT WE WERE INTERESTED
TO HAVE DATA RELATIVELY FAST
THAT COULD INFORM IN THE
ADOLESCENT YEARS.
THAT IS WHY WE DID NOT START THE
INFANCY.
SO WE SELECTED 21 SITES ACROSS
THE UNITED STATES, ALL OF WHICH
BASICALLY HARMONIZED THEIR BRAIN
IMAGING PROTOCOLS, THEIR
ASSESSMENTS, BEHAVIORAL AND
COGNITIVE ASSESSMENTS, SOCIAL
ASSESSMENTS AND ENSURING THERE
WAS OVERREPRESENTATION OF
UNDERREPRESENTED GROUPS.
THIS WAS IMPORTANT BECAUSE IF WE
RELY JUST ON DIRECT PRESENTATION
OF 12% OF AFRICAN-AMERICANS OR
15% OR 17% OF HISPANICS, WE
WOULDN'T HAVE A SUFFICIENTLY
LARGE SAMPLE TO ADDRESS HOW
FACTORS ASSOCIATED WITH RACE AND
ETHNICITY MAY BE INFLUENCING THE
MEASURES WE WERE LOOKING FOR.
THE PROTOCOL ITSELF IS QUITE
INTENSIVE.
EVERY TWO YEARS ALL OF THE
PARTICIPANTS, CLOSE TO 12,000,
11,870, ACTUALLY EVERY TWO YEARS
THEY UNDERGO A IN DEPTH BRAIN
ASSESSMENTS, NOT JUST STRUCTURE
BUT FUNCTION.
FUNCTION AT RISK AND WITH
VARIOUS COGNITIVE AND SOCIAL
TASKS.
AND THEN EVERY YEAR THEY HAVE AN
EVALUATION ASSOCIATED WITH THIS
BRAIN IMAGING.
EVERY YEAR COGNITIVE
ASSESSMENTS, ASSESSMENTS FOR
HEALTH, ASSESSMENTS FOR SOCIAL
INTERACTIONS.
ASSESSMENTS FOR THE FAMILY TO
UNDERSTAND WHAT MAYBE BE
SOCIOECONOMICAL CHALLENGES THEY
HAVE.
THE LATTER, OTHER THAN FOR THE
BRAIN IMAGING AND BIOSPECIMENS
COLLECTED EVERY TWO YEARS ARE
DONE ON A YEARLY BASIS.
EVERY SIX MONTHS THEY CAN GET
ADDITIONAL SCREENING IF IT IS
NECESSARY AS I WILL COMMENT
LATER ON WHAT THE CASE IS WHEN
WE HAD THE COVID PANDEMIC.
SO THIS IS WHAT WE DID.
SOME OF THE TARGET AREAS WE WERE
LOOKING FOR.
A KEY TARGET THAT FACILITATED
BRINGING THE DATA TOGETHER ONCE
THAT WAS GOING TO BE AN OPEN
ACCESS STUDY.
SO FROM THE BEGINNING WE PAID
ENORMOUS ATTENTION, NOT JUST TO
MAKE THE DATA ACCESSIBLE AND
AVAILABLE AS FAST AS WE COULD
AND PLACES IT IN A DATA BASE AND
PROVIDING THE RESOURCES SO THAT
RESEARCHERS AND INVESTIGATORS
FROM ANY PLACE IN THE WORLD CAN
TAKE THESE DATA AND HAVE ACCESS
TO ANALYTIC AND PREANALYZED DATA
FOR THOSE THAT DON'T HAVE THE
COMPUTER CAPABILITIES SO THAT
THEY CAN ASK SCIENTIFIC
QUESTIONS THAT MAY BE OF
INTEREST FOR THEM.
OF COURSE, THIS IS EASIER SAID
THAN DONE BECAUSE THERE ARE
MAJOR CHALLENGES OF ISSUES OF
ENSURING CONFIDENTIALITY.
THERE WERE WE LEARNED MAJOR
CHALLENGES OF ASSURANCES THAT
THE DATA IS NOT UTILIZED FOR
NEGATIVE PURPOSES.
HOW DO YOU OVERSEE THAT?
BUT OVERALL, AS A RESULT OF
THAT, AND THE FACT THAT THE DATA
IS EXTRAORDINARILY COMPLEX, IT
HAS BASICALLY BEEN -- WE HAVE
BEEN FORCED TO CHANGE THE PLACE
WHERE WE PUT OUR REPOSITORIES.
THE DATA RELEASES WE HAVE ONLY
HAVE BEEN ABLE TO GO COMPLETELY
FOR FOUR YEARS FOLLOW-UP.
IN JANUARY 2025 WE DO THE DATA
RELEASE 6.0 THAT WILL FOLLOW FOR
SIX YEARS INFORMATION FROM THE
IMAGING TO THE BEHAVIORAL AND
SOME OF THE GENETIC DATA.
IN PARALLEL TO OPEN ACCESS FOR
DATA, WE HAVE BEEN RELEASING THE
BIOSPECIMENS AND THE
BIOSPECIMENS COLLECTING INCLUDE
HAIR, BLOOD, SALIVA.
ALL OF THIS IS LINKED WITH
GEOSPATIAL DATA WHICH, OF
COURSE, INCREASES RICHNESS WHICH
CAN BE SEEN IN THE NEXT SLIDE.
CAN I HAVE THE NEXT SLIDE.
NEXT SLIDE, GUYS.
HELLO.
YES.
SO THESE GIVES US A DIAGRAM OF
WHAT WE HAVE BEEN DOING 2016 TO
2024.
I WANT TO NOTE 96% OF THE ABCD
PARTICIPANTS HAVE BEEN RETAINED
EIGHT YEARS INTO THE STUDY.
I WANT YOU TO THINK ABOUT WHAT
THIS MEANS IN TERMS OF HOW WELL
OVERSEEN AND MANAGED THIS STUDY
HAS BEEN.
AND HOW ENGAGED THE PARTICIPANTS
THEMSELVES HAVE BEEN IN BEING
PART OF THE STUDY.
IT HAS EVALUATED FROM THE
CLASSICAL PHYSICAL HEALTH TO THE
PSYCHIATRIC AND MENTAL AND
COGNITIVE AND SOCIAL
INTERACTIONS AND BEHAVIORS OF
THESE CHILDREN.
THIS WHEN YOU LINK WITH GEOCODED
DATA HAS PROVIDED THE MOST UP TO
DATE IN DETAIL LARGE EVALUATION
OF SOERNL DETERMINANTS OF HEALTH
AND HOW IT INFLUENCES THE BRAIN,
MENTAL ILLNESS, SUBSTANCE USE
DISORDER AND OTHER MEDICAL
CONDITIONS.
LET ME GIVE YOU SOME EXAMPLES OF
THIS.
THE FIRST STUDY, SORRY, BEFORE I
JUMP INTO THE EXAMPLES
THEMSELVES, IN TERMS OF WHAT WE
ARE EIGHT YEARS LATER, ALWAYS
LOOK AT METRICS.
BUT IN TERMS OF PUBLICATIONS AND
THE DATA HAS BEEN RELEASING AS
WE ARE GATHERING IT, AS OF NOW
THERE HAVE BEEN MORE THAN 1,150
PUBLICATIONS.
WHAT YOU SEE THERE IS A
CUMULATIVE -- SO THEY HAVE BEEN
INCREASING EVERY SINGLE YEAR.
MORE AND MORE RESEARCHERS ARE
JUMPING INTO THE DATA.
AGAIN, THE RESULTS ARE FROM ALL
OVER THE WORLD, THE
INVESTIGATORS THAT ARE
PUBLISHING IN DATA.
WE HAVE IN BLUE INDIVIDUALS THAT
ARE PART OF THE ABCD WHO ARE THE
AUTHORS OF THESE PAPERS AND IN
ORANGE ARE INDIVIDUALS THAT ARE
NOT DIRECTLY OR INDIRECTLY
ASSOCIATED WITH THE ABCD STUDY
BUT TAKEN ADVANTAGE OF THE OPEN
ACCESS OF THE DATA SETS AND THE
CAPABILITIES WE OFFER WITH IT TO
ENSURE THAT THE DATA IS BEING
USED.
ALSO THE ABCD HAS NOT JUST
INSPIRED A LOT OF WORK AND
ADVANCES, BUT THEY HAVE ALSO
INSPIRED OTHER RESEARCHERS AND
TO DO SPECIFIC SCIENTIFIC, TO
FOLLOW SPECIFIC SCIENTIFIC
QUESTIONS.
AND THE ABCD HAS BEEN USED TO
ACTUALLY AS A PLATFORM TO
ACTUALLY PROPOSE SPECIFIC
PROTOCOLS AND THEY ARE FUNDED
THROUGH GRANTS OF MULTIPLE
INSTITUTES.
OF THOSE INSTITUTES THE NIMH HAS
FUNDED MOST SATELLITE GRANTS,
45% OF THEM, BECAUSE IT IS
NATURALLY A VERY, VERY RELEVANT
TOPIC FOLLOWED BY NIDA.
LET ME JUST GET YOU, AGAIN, THE
EXAMPLES THAT I WAS PROMISING
BEFORE.
EXAMPLE NUMBER ONE, AND I CHOSE
IT BECAUSE I THINK -- NEXT
SLIDE.
I THINK ABOUT THE ABCD AND TO ME
A PSYCHIATRIST WORKING IN THE
BRAIN, MOST CONSEQUENTIAL IMPACT
OF THE ABCD STUDY HAS BEEN TO
ILLUMINATE HOW THOSE SOCIAL
DETERMINANTS OF HEALTH FACTORS
TREMENDOUSLY INFLUENCE THE
DEVELOPMENT OF THE HUMAN BRAIN.
AND THIS STUDY WHICH IS THE
FIRST ONE TO DOCUMENT THAT WAS
PUBLISHED IN 2020.
THEY WERE LOOKING AT THE EFFECTS
OF LEAD EXPOSURES IN BRAIN
DEVELOPMENT.
THIS IS VERY RELEVANT BECAUSE IT
HAS BEEN WELL RECOGNIZED FOR
MANY, MANY YEARS THAT LEAD IS
TOXIC AND CHILDREN THAT GET
EXPOSED TO LEAD IN THEIR HOUSES
ARE ACTUALLY PERFORMING WORSE
COGNITIVELY AT SCHOOL.
INVESTIGATORS TOOK ADVANTAGE OF
THE ABCD AND ACTUALLY ASSESSED
LEAD EXPOSURES WERE R ASSOCIATED
WITH COGNITIVE PERFORMANCE ON
THESE CHILDREN AND THAT IS WHAT
YOU SEE IN THE TOP DIAGRAM WHERE
YOU ACTUALLY CAN SEE ON THE X
AXIS THE LEVELS OF LEAD EXPOSURE
AND Y AXIS, PERFORMANCE, HIGHER
NUMBERS IN THE Y AXIS FOR
COGNITIVE PERFORMANCE, BUT YOU
ARE DIVIDING THE ABCD EXAMPLE IN
THREE SUBGROUPS.
THE THREE SUBGROUPS ARE DIVIDED
ON THE BASIS OF THE FAMILY
INCOME OF THE CHILDREN.
CHILDREN WITH LOW INCOME, MIDDLE
INCOME AND WITH HIGH INCOME.
AND WHAT THEY NOTED IS CHILDREN
THAT GET EXPOSED TO HIGH LEVELS
OF LEAD DO HAVE LOWER COGNITIVE
PERFORMANCE.
WHAT IS NOT IMMEDIATELY JUMPS AT
YOU LOOKING AT THAT GRAPH IS
THAT IT IS NOT -- IT IS THE
CHILDREN THAT COME FROM LOW
INCOME YOU SEE THE NEGATIVE
EFFECTS OF LEAD ON COGNITIVE
PERFORMANCES.
THE LOWER DIAGRAMS SHOW TWO
MEASURES OF THE BRAIN THAT
ACTUALLY ARE INDICATORS OF BRAIN
DEVELOPMENT THAT IS ON THE ONE
HAND THE VOLUMES OF DIFFERENT
AREAS OF THE BRAIN AND THE OTHER
IS THE CORTICAL SURFACE.
AT LOT OF COGNITIVE OPERATIONS
ARE RELATED TO EXPANDED --
SORRY, THE LEFT IS THE CORTICOL
SURFACE AND THE RIGHT IS THE A
EXPOSURE.
THIS WAS OBSERVED ONLY ON THE
CHILDREN OF LOW INCOME.
IT DOES OF DIRECT POLICY
RELEVANCE BECAUSE IT INDICATED
THAT, YES, ENVIRONMENTAL
EXPOSURES ARE NEGATIVE.
THERE ARE NO TWO WAYS AROUND IT.
RIGHT NOW WE ARE VERY INTERESTED
IN LOOKING AT WHETHER EXPOSURES
WE CAN MONITOR IN THESE CHILDREN
ARE GOING TO BE ASSOCIATED WITH
ANY CHANGES OR NO?
IT ALSO HIGHLIGHTS THAT INDEED
THERE ARE INTERACTIONS BETWEEN
THESE EXPOSURES AND AN EFFECT
THAT IF YOU HAVE AN INSULT, YOU
CAN BUMP AND COMPENSATE.
BUT IF YOU START TO HAVE
COMMUNITY INCIDENTS OF THOSE
INSULTS, THAT IS WHERE YOU START
TO IMBALANCE THE SYSTEM AND THAT
IS SHOWING IN THE CASE OF THE
INDIVIDUALS THAT ARE COMING FROM
LOW INCOME AND MUCH LESS LIKELY
TO BE ABLE TO THE EXPOSURE TO
ADVERSE EFFECTS.
NEGATIVE EFFECTS OF LEAD AND
NEGATIVE EFFECTS OF INCOME.
SOCIOECONOMIC DEPRIVATION, LOW
INCOME CAPABILITIES IS UP WITH
OF THE MOST IMPORTANT FACTORS
THAT WE ARE OBSERVING
INFLUENCING THE BRAIN
TRAJECTORIES OF THESE CHILDREN.
THIS IS SHOWN ELOQUENTLY BY THIS
STUDY THAT WAS PUBLISHED LAST
YEAR BECAUSE IT GOES BEYOND THE
NOTION OF, YES, WE KNOW THAT
FAMILIES FROM LOW INCOME, THOSE
CHILDREN ARE GOING TO HAVE
BRAINING WITH SMALLER VOLUMES,
SMALLER CORTICOL SERVICES AND
CORTICOL THICKNESS.
WHAT THESE INVESTIGATORS DID WAS
SOMETHING VERY CLEVER.
THEY SAID WE HAVE 17 DIFFERENT
STATES WHERE WE ARE SCREENING
AND RECRUITING AND TESTING THESE
CHILDREN.
THESE STATES DIFFERENCE VERY
MUCH IN COST OF LIVING.
SOME STATES ARE VERY, VERY
EXPENSIVE TO LIVE IN THOSE
STATES LIKE CALIFORNIA OR NEW
YORK.
OTHER STATES WHERE THE CENTERS
ARE RECRUITING HAVE VERY LOW
COSTS OF LIVING.
YOU MAY HAVE YOUR INCOME, BUT IT
IS AFFECTED BY YOUR CONTEXT.
SO THEY LOOK AT THAT, THE COST
OF LIVING IN THE STATE WHERE
CHILDREN WERE BEING RECRUITED
AND LOOK AT THE SECOND PARAMETER
IN EACH STATE.
WHAT WAS THE LEVEL OF THE
AVERAGE CASH BENEFIT ON EACH ONE
OF THESE STATES TO THE FAMILIES
SUCH THAT STATES COULD HAVE HIGH
COST OF LIVING BUT HIGH AVERAGE
CASH BENEFITS.
AS OPPOSED TO STATES THAT HAVE
VERY HIGH COST OF LIVING BUT
VERY LOW CASH BENEFITS.
AND THEY ASKED THE QUESTION IN
AVERAGE HOW DOES THAT DIFFERENCE
BETWEEN THE COST OF LIVING AND
THE ASSOCIATE OF COST OF LIVING
WITH AVERAGE CASH BENEFITS
INFLUENCE THE PSYCHOPATHOLOGY OF
THESE CHILDREN?
WHAT YOU SEE IS INTERNALIZING
PROBLEMS OF CHILDREN COMING FROM
STATES THOSE FOUR
CLASSIFICATIONS.
AND ALSO THEY LOOK AT THE BRAIN
AND THE BRAIN VOLUMES.
AND THEY SHOW INDEED THAT
CHILDREN THAT COME FROM LOW
INCOME FAMILIES REGARDLESS OF
THEIR BEING IN A HIGH COST OF
LIVING WITH HIGH SYMPTOMS.
YOU SEE HERE AS THE STEEPLE
BARS.
CHILDREN FROM HIGH INCOME, IT
DOESN'T MATTER WHERE YOU COME
FROM.
YOU BASICALLY HAVE LOWER LEVELS
OF INTERNALIZING SYMPTOMS.
HOWEVER, CHILDREN FROM LOW
INCOME THAT CAME FROM HIGH CASH
BENEFITS HAD SIGNIFICANTLY LOWER
LEVELS OF INTERNALIZING SYSTEMS
THAT THOSE CHILDREN OF LOW
INCOME THAT CAME FROM STATES OF
LOW AVERAGE CASH BENEFIT.
HOW LARGE WAS THAT DIFFERENTIAL?
48% REDUCTION IN INTERNALIZING
SYMPTOMS IN THOSE CHILDREN OF
LOW INCOME THAT CAME FROM HIGH
CASH BENEFITS.
AND SIMILARLY, THE REDUCTION IN
TERMS OF THE DEVELOPMENTAL
EFFECTS WAS A 34% REDUCTION
HIPPOCAMPAL VOLUME DISPARITIES
TO MAKE A SIGNIFICANT DIFFERENCE
IN OVERALL BRAIN DEVELOPMENT
COGNITION AND PSYCHOPATHOLOGY.
THIS IS IMPORTANT BECAUSE AS WE
ARE SPEAKING ABOUT PREVENTION,
SPEAKING ABOUT DECISIONS AND
POLICIES AND INTERVENTIONS,
BEING ABLE TO HAVE DATA LIKE
THIS CAN GUIDE THE STATES TO
MAKE -- TO TAILOR THEIR
INTERVENTION.
THESE ARE TWO EXAMPLES.
BOTH OF THEM RELATED TO
SOCIOECONOMIC FACTORS TO
ENVIRONMENTAL EXPOSURES AND THEN
THE THIRD EXAMPLE I WANT TO GIVE
YOU WHICH IS IN THE NEXT SLIDE
IS ACTUALLY ONE THAT ALL
PSYCHIATRISTS HAVE BEEN
SALIVATING FOR MANY, MANY YEARS
WHICH IS THE NOTION OF HOW DO WE
IDENTIFY A CHILD THAT IS AT HIGH
RISK FOR A PSYCHIATRIC DISORDER?
WE TEND TO DO THAT, OF COURSE,
IF YOU HAVE A FAMILY HISTORY.
IT IS NOT SO SIMPLE.
AS A RESULT OF THAT, THE SENSE
IS WHEN CHILDREN OR ADOLESCENTS
START TO SHOW PSYCHOPATHOLOGY
THEY ARE ALREADY QUITE ADVANCED.
LIKE ANY OTHER DISEASES CAN WE
INTERVENE AS EARLY AND POSSIBLE
TO PRET THOSE TRAJECTORIES THAT
CAN LEAD TO A SEVERE MENTAL
ILLNESS.
THERE HAS BEEN AN ENORMOUS
AMOUNT OF WORK IN PSYCHIATRY TO
USE IMAGING, ALSO POLYGEN
RESCORES AND BRAIN IMAGING TO
IDENTIFY BIOMARKERS ASSOCIATED
WITH MENTAL ILLNESS.
RECENTLY A VERY INTERESTING
STORY WAS ACTUALLY REPORTED.
THIS WAS IN 2024, WHICH YOU SEE
THE IMAGES TO THE LEFT THAT IN
THE FIRST COLUMN IS CONTROLLED
INDIVIDUALS.
AND THESE ARE BRAIN, SAGITAL
BRAIN IMAGES THE UPPER IS
LATERAL AND TORE IS MIDLINE.
IN LATERAL AND MIDLINE YOU
IDENTIFY THE REGIONS IN BLACK
REPRESENT THE SALIENT NETWORK
THAT IS THE VALUE OF A
PARTICULAR STIMULANT.
WHAT THESE SHOW AND COLLABORATE
IN EACH COHORT WHICH EACH COLUMN
IS A REPLICATION, INDIVIDUALS
SUFFERING FROM DEPRESSION HAD AN
EXPANSION, THAT IS THE THE
SURFACE AREA OF THE SALIENT
NETWORK IS MUCH LARGER OF THE
PATIENT, OF THEIR CORTEX IS MORE
LARGER THAN IN CONTROLS.
THIS WAS REPLICATED IN THREE
DIFFERENT SAMPLES, LARGE SAMPLES
OF INDIVIDUALS WITH DEPRESSION.
RESEARCHERS WENT ON AND SAID
WHAT ABOUT IF WE LOOK AT THE
LONGITUDINAL DATA FROM THE ABCD,
IDENTIFY CHILDREN AND THERE IS
AN EXPANDED REPRESENTATION OF
THE SALIENT NETWORK IN THE BRAIN
CAN WE USE THAT AS THE PREDICTOR
OF DEPRESSION.
THEY ARE ABLE TO SHOW THEY CAN
PREDICT DEPRESSION SYMPTOMS ON
THE BASIS OF THE SALIENCY
NETWORK.
CHILDREN WHO WILL BE DEVELOPING
DEPRESSION SYMPTOMS, TEND TO
HAVE A LARGER REPRESENTATION OF
THE SALIENT NETWORK.
THIS IS AN EXAMPLE THIS ENABLES
TO IDENTIFY BIOMARKERS TO HELP
GUIDE EARLIER INTERVENTIONS.
THESE ARE THREE EXAMPLES I PICK
UP BECAUSE, OF COURSE, I LOVE
THE BRAIN AND EXTREMELY
INTERESTED IN HOW IT UNDERLIES
OUR BEHAVIORS AND
PSYCHOPATHOLOGY, BUT IT IS ALSO
VERY IMPORTANT REGARDING
POLICIES AND GUIDELINES.
NEXT SLIDE.
SO NOT SURPRISING BECAUSE THIS
IS A STUDY THAT HAS JUST BEEN
DISSEMINATED EVERYWHERE, IT HAS
HAD A TREMENDOUS IMPACT
VIS-A-VIS QUALITY DOCUMENTS AND
CLINICAL AROUNDS THE WORLD.
THE UNITED NATIONS, THE FRENCH
GOVERNMENT, THE COLUMBIAN
GOVERNMENT, THE BRITISH
GOVERNMENT.
IT IS RECOGNIZED HOW IT CAN HELP
US IDENTIFY ASPECTS THAT IN THE
PAST WERE NOT OBSERVABLE.
THE NEXT SLIDE IS ONE THAT I
SHOWED, THE NEXT SLIDE, PLEASE,
BECAUSE IT ACTUALLY DOES IN A
VERY CONCRETE WAY EXEMPLIFIES
ONE OF THOSE GUIDELINES AND THE
GUIDELINES RECENTLY PUBLISHED
FORKED A ADHD.
WHY THIS IS IMPORTANT IS ADHD IS
STILL QUESTIONED BY MANY ABOUT
IT BEING A REAL DISORDER.
IT IS BEING QUESTIONED WHAT IS
THE OPTIMAL TREATMENT AND HOW TO
RECOGNIZE IT.
TO BE ABLE THROUGH THE ABCD
STUDY WHICH APPROXIMATELY 10% OF
THOSE CHILDREN, 8% TO 10% HAVE A
DIAGNOSIS OF ADHD ALLOWS US TO
DETERMINE IF TREATMENT SOMETIMES
QUESTIONED BY OTHERS IS
ASSOCIATED WITH IMPROVEMENTS IN
BRAIN DEVELOPMENT, IN FUNCTION,
AND/OR ADDRESS THAT IMPORTANT
QUESTION THAT SOMEONE HAS
BROUGHT UP, COULD IT MAKE THEM
MORE VULNERABLE TO THE USE OF
SUBSTANCES GATHERING DATA AND
EVIDENCE HAS BEEN TREMENDOUSLY
POWERFUL TO ADVANCE THESE
FIELDS.
AS WE LOOK INTO WHAT WE ARE
OBSERVING, THERE ARE MANY
CHANGES HAPPENING IN THE
LANDSCAPE OF ADOLESCENTS.
AND SORT OF THIS HAS GENERATED A
LOT OF CONCERN IN GENERAL FROM
OUR SOCIETY.
FOR EXAMPLE, SLEEP.
I MEAN, WE HAVE THE WHOLE ALL
ALONG VERY MUCH CONCERNED ABOUT
THE NOTION THAT SLEEP IS CRUCIAL
FOR BRAIN DEVELOPMENT.
WE KNOW THAT AND SO THE
RECOMMENDATIONS CHILDREN SHOULD
SLEEP 8 TO 10 HOURS A DAY AND
THE ABCD DATA SAYS THAT IS NOT
HAPPENING VERY MUCH IN THE
UNITED STATES.
APPROXIMATELY 48% OF THOSE
CHILDREN ARE ACTUALLY SLEEPING
THE RECOMMENDED HOURS.
RESEARCHERS ARE STARTING TO LOOK
AT HOW THAT AMOUNT OF SLEEP THAT
ADOLESCENTS HAVE INFLUENCES
THEIR BRAIN DEVELOPMENT AND HOW
INTERESTINGLY WHAT THE STUDIES
ARE SHOWING IS TREMENDOUS
DISPARITIES IN SLEEP SUCH THAT
BLACK AMERICANS, FOR EXAMPLE,
ADOLESCENTS SLEEP HALF AN HOUR
LESS THAN WHITE AMERICANS.
INDIVIDUALS ON THAT, THE MAIN
CONTRIBUTOR OF THAT ACTUALLY
REFLECTS DIFFERENCES IN
SOCIOECONOMIC, LOWER INCOME IN
THE FAMILIES OF BLACK AMERICANS
DRIVES MANY OF THESE
DIFFERENCES.
ANOTHER EXTREMELY IMPORTANT ONE
AND I WOULD SAY ONE WE NEED TO
ACTUALLY GOING TO BE ABLE TO
UNDERSTAND HOW IT INFLUENCES
ADOLESCENCE IS SOCIAL MEDIA.
THERE IS TREMENDOUS CONCERN THAT
THE OVERUTILIZATION OF SOCIAL
MEDIA ON THE ONE HAND HAS MADE
ADOLESCENTS LESS LIKELY TO
UNDERSTAND AND INTERACT WITH
PEERS IN WAYS THAT LEAD TO
PLAYFULNESS AND PHYSICAL
ACTIVITIES AND FACILITATES
BULLYING, SUICIDE, DEPRESSION.
UNDERSTANDS HOW SOCIAL MEDIA
INFLUENCES BEHAVIORS, NETWORKS
IN THE BRAIN IS CRUCIAL AND THE
ABCD HAS BEEN FOCUSED TO
UNDERSTAND THESE QUESTIONS.
WE HAVE AN INFOGRAPHIC WHERE WE
SUMMARIZE SOME OF THE RESULTS.
IT SHOWS TEENAGERS WHO USE
SOCIAL MEDIA EXTENSIVELY HAVE
HIGHER LEVELS OF DEPRESSION,
ANXIETY.
IT SHOWS IN SOME INSTANCES SOME
OF THE USE OF SOCIAL MEDIA CAN
HAVE BENEFITS.
AND THERE WAS A PARTICULAR PAPER
THAT WAS VERY INTERESTING
BECAUSE IT IS SHOWING THAT IN
TEENAGER THAT PLAY VIDEO GAMES
FOR EXCESSIVE HOURS, FOR MORE
HOURS TEND TO HAVE BETTER
WORKING MEMORIES AND REACTION
PAST THOSE THAT DO NOT.
UNDERSTANDING HOW SOCIAL MEDIA
AFFECTS ADOLESCENTS IS CRUCIAL.
THE ABCD WAS STARTED IN 2016.
WE STARTED COLLECTING DATA THEN.
IN 2020, WE HAVE A -- AND THEN
THERE WERE SOCIALIZATION AND
SCHOOL CLOSURES AND THE
INTERACTION OF TEENAGERS CHANGED
COMPL
COMPLETELY.
THEIR EDUCATION CHANGED BECAUSE
OF COVID.
SO VIEWING THE OPPORTUNITY WE
HAD IN 2020, 2021, WE BASICALLY
DID SUPPLEMENTAL FUNDING TO ABCD
INVESTIGATORS TO ENSURE THEY
WERE COLLECTING DATA THAT WAS
RELEVANT INCLUDING THE ISOLATION
WAS INFLUENCING THESE TEENAGERS.
ALSO INCLUDING DATA TO ASSESS
HOW THE FAMILY ENVIRONMENT
ITSELF WAS BEING INFLUENCED BY
THE COVID PANDEMIC AND, OF
COURSE, IMPORTANTLY WHERE THEY
WERE BEING INFECTED IN THEIR
FAMILY OR NOT OR NEGATIVELY
AFFECTED BY THE CONSEQUENCES OF
COVID.
SEVERAL PAPERS HAVE ALREADY BEEN
PUBLISHED ON THIS, BUT I WOULD
SAY IMPORTANTLY, FOR EXAMPLE, WE
HAVE THE DATA LONGITUDINAL OF
THOSE TEENAGERS THAT GOT
INFECTED WITH COVID AND THOSE
THAT MOST OF THEM BY NOW HAVE.
OF THE TEENAGERS THAT GOT
VACCINATED VERSUS THOSE THAT DO
NOT.
THE DATA IN TERMS OF HOW THESE
TEENAGERS, WHETHER THEY DO
DEVELOP LONG LASTING SYMPTOMS OF
COVID AND HOW THAT INFECTION, IF
AT ALL, INFLUENCES THEIR BRAIN.
IT IS OF IMPORTANCE BECAUSE WE
HAVE KNOWN OF VIRAL INFECTIONS
THAT HAVE BEEN ASSOCIATED WITH
PSYCHIATRIC SYMPTOMS.
THIS IS JUST TO THANK EVERYONE.
THERE ARE 10 FEDERAL PARTNERS
AGENCIES INVOLVED, 21 RESEARCH
SITES, HUNDREDS OF INVESTIGATORS
AND TRAINEES, EVEN MORE RESEARCH
ASSISTANTS AND STAFF.
CLOSE TO 12,000 PARTICIPANTS AND
THEIR FAMILIES.
I DO WANT TO THANK -- WHO HAS
BEEN AN EXTRAORDINARY LEADER FOR
THIS STUDY.
THANKS VERY MUCH FOR YOUR
ATTENTION AND I'M HAPPY TO
ANSWER ANY QUESTIONS IF THERE
ARE ANY.
>> THANKS, VERY MUCH, NORA.
COULD WE PULL DOWN THE SLIDES,
PLEASE, AND LET'S SEE WHO WOULD
LIKE TO MAKE THE COMMENT OR ASK
A QUESTION.
>> I THINK YOU OVERWHELMED
EVERYBODY.
>> I GUESS SO.
>> WAFAA, PLEASE.
>> THANK YOU FOR REMARKABLE
PRESENTATION AND IMPORTANT WORK.
I SEE YOU MUST HAVE DATA ON
THIS.
HOW MUCH ARE YOU CONNECTING
INFORMATION ON THE FAMILIES?
SIBLINGS AND FAMILY MEMBERS OF
THE, I GUESS THE ADOLESCENTS IN
THE COHORT AS THEY MOVE FORWARD?
>> YES.
I MEAN, IT IS VERY IMPORTANT TO
UNDERSTAND THAT INDIVIDUAL
WITHIN THE COHORT OF THEIR
FAMILY.
SO THERE IS DETAILED INFORMATION
OF THE FAMILY STRUCTURE AND ITS
CHARACTERISTICS.
AS IMMEDIATE FAMILY AND FURTHER
APART FAMILY AS WELL AS FRIENDS
AND SOCIAL NETWORKS.
SO, YES, INDEED, PARTICULARLY
RELEVANT AS I WAS MENTIONING FOR
THE COVID PANDEMIC WAS
RECOGNIZING HOW FOR EXAMPLE THE
PARENTS WERE FACING THE COVID
PANDEMIC.
DID THEY LOSE THEIR JOB?
DID THEY HAVE A SAFE PLACE WHERE
THEY COULD BE?
DID THEY HAVE ENOUGH RESOURCES?
AND ALL OF THESE FACTORS HAVE
BEEN FOLLOWED UP TO INDICATE
THAT THE LEVEL OF SOCIAL STRESS
IN THE FAMILY PREDICTED WHETHER
THE TEENAGER WILL SHOW SYMPTOMS
OR NOT DURING THE COVID
PANDEMIC.
THE MOST IMPORTANT AVAILABLE
THAT CAN PREDICT HOW TEENAGERS
WERE RESPONDING TO THE COVID
PANDEMIC THEMSELVES.
>> THANK YOU.
SO TO KEEP ON TIME, BRIAN, WE'RE
GOING TO LET YOU HAVE THE LAST
WORD, PLEASE.
>> THARNGS.
NORA, SO EXCITING TO SEE THE
UPDATE ABOUT THE ABCD STUDY.
AS THE TEENAGER ARE NOW 2024,
2026 REACHING LATER R TEENAGE
YEARS.
ARE YOU PLANNING TO DO HIV
TESTING?
IT SEEMS THERE IS AN OPPORTUNITY
TO LOOK AT PATTERNS AND
TRAJECTORIES THAT MIGHT PUT THEM
AT RISK FOR HIV PARTICULARLY
INDIVIDUALS AT HIGHER RISK?
>> DEFINITELY.
BECAUSE THE STUDY WILL BE ENDING
IN 2027 AND IT IS IN THE
TRANSITION WHERE YOU START TO
SEE THE EMERGENCE OF RISKIER
BEHAVIORS.
IN THE ABCD 2.0, TO FOLLOW THEM
AND UNDERSTAND HOW IT INFLUENCES
THEIR TRAJECTORIES.
YES, THEY COULD GET OTHER
DISEASES LIKE HIV, BUT OTHER
SEXUALLY TRANSMITTED DISEASES
THAT ARE QUITE PREVALENT AS WELL
AS MEDICAL CONDITIONS.
IT WAS CLEAR I WAS FOCUSING ON
MENTAL ILLNESS AND SUBSTANCE
USE, BUZZ THAT STAGE OF LIFE CAN
CEMENT BEHAVIORS THAT INFLUENCE
DIABETES, OR HYPERTENSION.
SO, YES, INDEED.
>> THANK YOU.
>> THANKS VERY MUCH.
BACK TO YOU, MONICA.
>> THANK YOU FOR MUCH DR. VOLKOW
FOR THAT AMAZING PRESENTATION
AND SO MUCH MORE TO COME FROM
ABCD.
NEXT WE ARE GOING TO MOVE ON TO
HELA GENOME DATA ACCESS WORKING
GROUP FOR SOME DISCUSSION ON
DATA ACCESS.
DR. LYRIC JORGENSON AND GARH
GRAHAM WHO IS THE DIRECTOR OF
GLOBAL HEALTH FOR HEALTH CARE
AND PUBLIC HEALTH AT
GOOGLE/YOUTUBE.
>> THANK YOU.
>> I'M GARTH GRAHAM AND I SERVE
ALONGSIDE LYRIC JORGENSON.
WE WILL REPORT ON THE EVALUATION
OF A RESEARCH PROPOSAL TO ACCESS
THE HELA SIGN GENOMIC DATA.
SO THE -- MANY OF YOU PROBABLY
KNOW THE GILA DATA USE AGREEMENT
HAS EXAMINATIONS FOR
EXPECTATIONS WISHING TO ACCESS
HELA CELL WHOLE GENOME SEQUENCE
DATA.
INVESTIGATORS ABIDE BY THE TERMS
SET FORTH IN THE AGREEMENT AND
TO USE THE DATA FOR SPECIFIC
PURPOSES, TO DISCLOSE IF THE
RESEARCH COULD RESULT IN A
PATENT OR COMMERCIAL PRODUCT.
AND RECOGNIZING THE
CONTRIBUTIONS OF THE LACKS
FAMILY FROM USING THESE HELA
CELLS AND DEPOSIT FUTURE GENOME
CELLS FROM THE HELA CELLS BACK
INTO THE DATA BASE.
NEXT SLIDE, PLEASE.
SO THIS IS THE CURRENT ROTHER OF
THE GILA WORKING GROUP WHOSE
WORK IS COORDINATED BY THE
EXECUTIVE SECRETARY, CHERYL
JACOBS.
AND WE ARE VERY, VERY GRATEFUL
TO HAVE HENRIETTA LACKS'
GRANDSON AND HER GRANDDAUGHTER
AND HER GREAT GRANDDAUGHTER
VERONICA ROBINSON WHO SERVED
ALONGSIDE THE REST OF US ON THE
WORKING GROUP.
SO AS A REMINDER, THIS IS THE
CRITERIA THE WORKING GROUP WHEN
EVALUATING AN ACCESS QUESTION.
THE HOLA DATA AGREEMENT ADVISED
THE INVESTIGATOR TO ADDRESS
THESE CRITERIA.
THE OBJECTIVE, DESIGN AND A
ANALYSIS AND AN EXPLANATION WHY
IT IS AVAILABLE FOR THE PROPOSED
RESEARCH.
ANY PLANS FOR IP OR
COMMERCIALIZATION AS YOU SEE
HERE OR PRODUCT SERVICE IS NOT
USED BY THE WORKING GROUP TO
MAKE A FINAL EVALUATION.
THIS IS MORE FOR DISCLOSURE
PURPOSES.
EVALUATION DOES NOT INCLUDE ANY
EVALUATION OF THE SCIENTIFIC
MERIT OF THE RESEARCH.
SO THIS SLIDE IS A QUICK
SNAPSHOT OF THE STATISTICS OF
THE REQUESTS THAT HAVE COME IN
THAT HAVE BEEN EVALUATED BY THE
WORKING GROUP AND APPROVED BY
THE NIH DIRECTOR.
AND THERE ARE INSTANCES WHERE
REQUESTS HAVE BEEN DISAPPROVED.
REQUESTS WILL BE DISAPPROVED IF
THEY DON'T MEET THE EXPECTATIONS
DESCRIBED IN THE HELA DATA USER
AGREEMENT.
WE WILL BE EVALUATING ONE
REQUEST THAT CAME IN AND
REPORTING THAT OUT HERE TO THE
ACD.
SO THIS REQUEST IS FROM THE
UNIVERSITY OF QUEENSLAND IN
AUSTRALIA AND AN EXAMPLE OF HOW
INVESTIGATORS ARE UTILIZING THE
HELA CELL DATA UTILIZED FROM THE
LACKS NIH AGREEMENT.
CONTAINS DATA THAT MOVE AROUND
OR JUMP.
RESEARCH ON JUMPING GENES HAVE
BEEN DONE USING HELA CELLS AND
THE HELA CELL GENOME.
THE STUDY THAT USED THE HELA
CELLS TO GENERATE HELA CELL
WHOLE GENOME SEQUENCE DATA TO
STUDY JUMPING GENE PREFERENCES.
THEY SUBMITTED THEIR HELA CELL
DATA TO THE GENOME SEQUENCES
STUDIES AS PART OF THE
AGREEMENT.
THE INVESTIGATOR IN THIS CASE
PROPOSES TO ACCESS THE GEE OHME
CELLS STUDIES SO THEY MAY ACCESS
THE HELA CELL DATA SUBMITTED BY
FLASH ET AL. REPORTED IN THE
2019 STUDY TO SEE FI THAT COULD
REPEAT OR REPLICATE THOSE
RESEARCHERS.
THAT CAME IN TO THE WORKING
GROUP.
WE USE A CRITERIA AS I
ARTICULATED EARLIER AND THE
WORKING GROUP FOUND THE REQUEST
FROM THE UNIVERSITY OF
QUEENSLAND TO BE CONSISTENT WITH
THE HELA DATA USE AGREEMENT.
AND SO NOW WE ARE GOING TO TURN
IT OVER THE THE ACD FOR ANY
FURTHER DISCUSSION.
>> OKAY.
THANK YOU VERY MUCH, GARTH.
I APPRECIATE THE VERY CLEAR
PRESENTATION.
AND SO FIRST I WANT TO KNOW IF
THERE ARE ANY COMMENTS FROM THE
ACD?
DO YOU NEED CLARIFICATION ABOUT
ANYTHING THAT GARTH PRESENTED
THIS MORNING?
YES, PLEASE, ELIZABETH.
>> I HAVE A BIT OF A SCIENTIFIC
QUESTION SINCE IT IS NEW TO ME,
THE HELA.
I KNOW THAT PEOPLE HAVE ALL
SORTS OF HELA CELL LINES THAT
HAVE DIVERTED FROM THE ORIGINAL
ONE.
IS THERE ANY EFFORT OR INTEREST
IN QUANTIFYING THIS?
I THINK HELA CELLS THAT ARE USED
BY DIFFERENT LABS TEND TO NOT BE
THE SAME.
IT WOULD BE GOOD TO TRY TO --
WHEN THEY HAVE BEEN PASSED A FEW
TIMES, CERTAINLY WHEN I WAS AN
ASSISTANT PROFESSOR I GOT ONE
FROM THE LAB NEXT DOOR AND STUFF
LIKE THAT AND WHEN PEOPLE
SEQUENCE THEM THERE ARE
INTERESTING JUMPS OR CHANGES IN
THEM.
IS THIS PART OF THE EFFORT?
AGAIN, I'M NOT VERY FAMILIAR
WITH IT, TO TRY TO SEE HOW MUCH
THESE CELLS HAVE DIVERGED OVER
TIME AND WHAT THAT MEANS FOR
SCIENTIFIC REASONS?
>> THANK YOU FOR THE QUESTION.
LYRIC, WOULD YOU LIKE TO --
>> SURE.
I'M HAPPY TO.
THAT IS A BIT OUTSIDE OF THE
SCOPE OF THE HELA WORKING GROUP.
FROM THE NIH PERSPECTIVE, THIS
IS SOMETHING WE CERTAINLY KEEP
TABS ON.
I CAN'T REMEMBER HOW MANY WE
HAVE REGISTERED WITH US, BUT WE
DO HAVE AN UNDERSTANDING OF
THOSE DIFFERENT VARIATIONS TO
YOUR POINT OVER TIME THE
PASSAGING HAS CHANGED SOME OF
THE SEQUENCING.
WE CAN SHARE THAT INFORMATION
WITH YOU AS FOLLOW-UP.
YES, PEOPLE ARE DEFINITELY
STUDYING IT.
>> ANY ADDITIONAL CLARIFICATIONS
THAT ARE REQUIRED BY ANYBODY?
OKAY.
SO I DON'T SEE ANY.
AND SO WHAT I WOULD LIKE TO DO
NOW IS TO ASK THE MEMBERS FOR A
MOTION TO PROCEED.
>> I MOVE TO PROCEED.
>> THANK YOU.
IS THERE ANY SECOND?
>> I'LL SECOND.
>> THANK YOU.
IS THERE FURTHER DISCUSSION
REQUIRED?
ALL RIGHT.
WE'LL DO THIS BY VOICE VOTE.
ALL THOSE IN FAVOR OF ACCEPTING
THE RECOMMENDATIONS FROM THE
COMMITTEE SAY AYE.
[ CHORUS OF AYES ]
>> ANY OPPOSED SAY NAY.
ANY ABSTENTIONS, PLEASE.
ALL RIGHT.
THANK YOU.
THE AYES HAVE IT.
AND THIS WILL BE TAKEN UNDER
ADVISEMENT BY THE NIH DIRECTOR.
LYRIC, TURNING IT TO YOU.
>> YES.
THERE ARE NEWER MEMBERS OF THE
ACD, THE WORKING GROUP THE NIH
HAS CREATED A DEDICATED WEBSITE
TO PUBLICATIONS FOR HELA CELLS
AND SOME OF THE ADVANCES MADE
FROM HELA CELLS.
I WILL SHARE THAT.
I WANT TO MAKE SURE NEWER
MEMBERS RECOGNIZE THAT IS A
COMPILATION BASED ON
RECOMMENDATIONS YOU ALL HAVE
MADE TO THE AGENCY.
>> THANK YOU.
THANK YOU TO THE COMMITTEE FOR
ITS GREAT WORK AND FOR THE
MEMBERS OF THE ACD.
AND BECAUSE OF YOUR EFFICIENCY,
YOU WILL GET ADDITIONAL BREAK
TIME NOW.
AND SO WE ARE GOING TO RECONVENE
AT 11:15.
SEE YOU ALL BACK AT 11:15. THANK YOU.
NOW WE ARE MOVING ON WITH THE
REST OF OUR SESSION TODAY AND WE
HAVE AN UPDATE FROM THE
ACCELERATING MEDICINES
PARTNERSHIP WITH THE FOUNDATION
OF THE NATIONAL INSTITUTES OF
HEALTH, JULIE GERBERDING, CHIEF
EXECUTIVE OFFICER IS HERE.
>> THANK YOU.
I KNOW HOW IMPORTANT AN ADVISORY
COMMITTEE TO THE DIRECTOR CAN BE
SO I APPRECIATE THE SERVICE OF
PEOPLE WHO ARE WORKING HARD TO
SUPPORT THE NIH.
I KNOW NOT EVERYONE KNOWS WHAT
THE FOUNDATION IS OR DOES, SO
I'M QUICKLY GOING TO GIVE YOU AN
INTRODUCTION.
THE FOUNDATION WAS CREATED BY
CONGRESS IN 1990 AND CAME INTO
EXISTENCE ABOUT SIX YEARS LATER.
SO WE'VE BEEN OPERATING TO
SUPPORT THE MISSION OF THE NIH
FOR ALMOST 30 YEARS.
IN THAT TIME, THE FOUNDATION HAS
GROWN FROM BEING I THINK AN
ORGANIZATION THAT MAINLY
PROVIDED LUNCH FOR NIH MEETINGS
TO ONE THAT HAS TAKEN AN
INCREASINGLY LARGE ROLE IN THE
SCIENTIFIC AGENDA, PARTICULARLY
IN THE TEAM SCIENCE ARENA.
I'VE BEEN LEADING THE FNIH FOR
JUST MORE THAN TWO YEARS, BUT IT
IS THE MOST JOYFUL JOB I HAVE
EVER HAD IN MY LIFE BECAUSE I'M
RIGHT UP CLOSE AND FRONT TO THE
INCREASE SCIENCE GOING ON AT THE
NIH.
NOT ONLY AT NIH, BUT LIFE
SCIENCE COMPANIES AND ACADEMIA.
IT IS AN AMAZING PRIVILEGE TO BE
PART OF THIS ORGANIZATION.
I'M GOING TO TRY TO JUST COVER
THREE THINGS TODAY, THREE
TAKEAWAY MESSAGES.
FIRST OF ALL, COLLABORATIVE TEAM
SCIENCE EXEMPLIFIED IN THE
ACCELERATED MEDICINE
PARTNERSHIPS WE ARE GOING TO
FOCUS ON IS ALIVE AND WELL AND
INCREDIBLY IMPORTANT TO OUR
ABILITY TO BRING
MULTISTAKEHOLDERS TOGETHER TO
SOLVE REALLY COMPLEX MEDICAL
PROBLEMS.
THE SECOND POINT IS THAT I
BELIEVE AND I THINK THIS IS WELL
SUBSTANTIATED, BUT TEAM SCIENCE
IS GOING TO BECOME MORE
IMPORTANT AS WE GO FORWARD
PARTICULARLY IN THIS ERA WHERE
EVERYBODY IS EXPERIENCING
CONSTRAINED RESOURCES, NOT JUST
GOVERNMENT, BUT ACADEMIA, THE
INDUSTRY, PATIENT ORGANIZATIONS,
AND ALL OF THIS CONFLUENCE MEANS
THE MORE WE CAN COLLABORATE AND
WORK TOGETHER THE MORE WE CAN
GET DONE AND I THINK THE BETTER
WE CAN GET IT DONE FOR FINITE
RESOURCES FOR SPECIFIC KINDS OF
PROBLEMS.
AND THEN LASTLY, I THINK FNIH IS
AN IMPORTANT PARTNER OF NIH.
OBVIOUSLY, WE ARE VERY SMALL
COMPARED TO THE SCOPE AND SCALE
OF NIH, BUT WE HAVE SOME
STRATEGIC LEVERAGE AND I THINK
THAT LEVERAGE REALLY IS A GOOD
VALUE FOR NIH AND OUR PARTNERS,
BUT I ALSO THINK IT IS A GREAT
VALUE FOR OUR TAXPAYERS WHO ARE
ULTIMATELY PAYING OUR BILL.
CAN I HAVE THE FIRST SLIDE,
PLEASE, JUST TO REMIND PEOPLE
HOW THIS ALL FITS TOGETHER.
YOU ALL KNOW THAT NIH HAS A
MISSION OF TURNING DISCOVERY
INTO HEALTH.
I'M NOT SEEING MY SLIDES SO I'M
NOT SURE.
ARE THEY UP, LARRY?
>> YES, THEY ARE.
>> ON THE NEXT SLIDE FADING INTO
HOW THE FNIH FITS INTO THAT.
WE AIM TO BE THE BRIDGE.
THAT IS WHY CONGRESS AUTHORIZED
US TO BRIDGE BETWEEN THE
GOVERNMENT WORK AND THE WORK
THAT THE PRIVATE SECTOR AND
OTHER PARTNERS CAN BRING TO THE
TABLE.
SO TRYING TO BUILD THESE BRIDGES
TO BREAK THROUGHS IN STRATEGIC
AREAS WHERE THEY CAN MAKE THE
MOST DIFFERENCES, REALLY, HOW WE
THINK ABOUT OUR WORK.
ON THE NEXT SLIDE I FRAME THAT
AS OUR ASPIRATION WHICH IS IN
CERTAIN CRITICAL AREAS TO REALLY
HELP AMPLIFY AND ACCELERATE THE
IMPACT OF NIH AND ITS
COLLABORATION.
WE DO THIS THROUGH THREE MAIN
ACTIVITIES.
ONE IS TEAM SCIENCE, WHICH I'M
GOING TO BE FOCUSING ON MOSTLY
IN THIS CONVERSATION.
BUT WE ALSO HAVE A SIGNIFICANT
PORTFOLIO FOR SUPPORT FOR
SCIENTISTS AND MORE RECENTLY
RECOGNIZING WE HAVE A VITAL ROLE
TO PLAY IN HELPING TO REEARN
TRUST IN SCIENCE.
SO LET ME START WITH THE TEAM
SCIENCE FRAMEWORK IN HOW WE
DECIDE TOGETHER WHEN IT MAKES
SENSE TO HAVE A PARTNER OR A
PARTNERSHIP SCIENCE,
PRIVATE-PUBLIC-PATIENT
PARTNERSHIP VERSUS THE MORE
TRADITIONAL KIND OF VERTICAL
MODEL OF RESEARCH SUPPORT.
THE MOST IMPORTANT FACTOR
OVERALL IS THERE A SIGNIFICANT
UNMET MEDICAL NEED IN NIH'S
WHEELHOUSE, BUT ALSO AN AREA
THAT IS UNIQUELY SUITED TO
PRIVATE-PUBLIC-PATIENT
PARTNERSHIPS.
AN AREA WHERE NIH CAN'T DO IT
ALONE, ACADEMIA CAN'T DO IT
ALONE, INDUSTRY CAN'T DO IT
ALONE.
IT TAKES THE COMBINATION OF THE
WISDOM AND CAPABILITIES, BUT
ALSO THE SCALE AND REACH.
SOMETIMES THAT IS PARTICULARLY
OBVIOUS IN TERMS OF POOLING DATA
SO THAT WE HAVE ENOUGH
STATISTICAL POWER TO DROP
INCLUSIONS OR PURSUE HYPOTHESIS.
OTHERS IS SKILL SET AND CROSS
FERTILIZATION OF PEOPLE WITH
DIFFERENT SCIENTIFIC
CAPABILITIES.
I THINK YOU GET THE PICTURE.
WE FOR PRACTICAL REASONS HAVE TO
MAKE SURE THIS MAKES SENSE TO
ALL THE INVOLVED PARTNERS.
THAT IT IS SOMETHING PEOPLE CARE
ABOUT.
THAT THERE ACTUALLY IS A VALUE
CREATED THROUGH THIS APPROACH
THAT IS PRECOMPETITIVE NOT
ANTI-COMPETITIVE FROM THE
PRIVATE SECTOR STANDPOINT.
WE HAVE BEEN BRAVE ENOUGH TO SAY
WE ARE PROCOMPETITIVE.
CREATES LONGITUDINAL DATA SETS
GENERATES HYPOTHESIS AND
STIMULATE DEVELOPMENT.
WE RECOGNIZE LEVERAGE IS
IMPORTANT AND IN THE NEXT COUPLE
OF SLIDES YOU'LL SEE HOW WE
LEVERAGE THE NIH INVESTMENT FROM
A VARIETY OF OTHER SOURCES SO WE
CREATE A POOL.
TO GIVE YOU A SENSE OF HOW THE
MONEY FLOWS WHEN WE CREATE ONE
OF THESE PARTNERSHIPS, I PUT
FNIH IN THE MIDDLE OF THIS, THE
FNIH CONTRIBUTION IS USUALLY
SMALL WITH RESPECT TO EVERYONE
ELSE'S CONTRIBUTIONS, BUT WE DO
RAISE MONEY FROM LIFE SCIENCE
COMPANIES, FROM LARGE AND SMALL
FOUNDATIONS AND PATIENT
ORGANIZATIONS AND INCREASINGLY
FROM PHILANTHROPISTES WHICH IS A
NEW DEVELOPMENT FOR US.
NIH APPROVES OUR MAJOR PROJECTS
AS DOES THE FNIH BOARD OF
DIRECTORS.
WHEN NIH IS PUTTING MONEY OUT
FOR ONE OF OUR COLLABORATIVE
PROJECTS, THEY DO IT USING THE
USUAL PEER REVIEW PROCESSES,
SUBJECT TO THE USUAL REGULATIONS
AND QUALITY ASSURANCES THAT
PROVIDES.
FNIH FUNDS ASPECTS OF THESE
PROJECTS AND WE MAKE OUR OWN
GRANTS AND CONTRACTS DIRECTLY.
OUR MAJOR ROLE IS TO DESIGN
THESE KIND OF COLLABORATIVE
FRAMEWORKS AND TO CONVENE AND
MANAGE THE OPERATIONAL
EXECUTION.
BUT THE GOVERNANCE OF THESE
EFFORTS IS SHARED.
NIH HAS A LARGE ROLE IN ENSURING
THE SCIENTIFIC QUALITY AS DO OUR
ACADEMIC AND ORR RESEARCH
PARTNERS PARTICULARLY AT THE
PROJECT LEVEL WHERE THE
GOVERNANCE IS LED BY SCIENTIFIC
EXPERTS IN A SPECIFIC FIELD.
THE NEXT SLIDE IS PRESENTED OVER
A FIVE-YEAR TIME FRAMEWORK WHICH
IS THE MOST RELEVANT TIME FOR
THE DISCUSSION AND THE SCALE AND
SCOPE OF THE FNIH IN ITS CURRENT
ITERATION.
THIS SHOWS YOU WHERE THE MONEY
COMES FROM OVER THAT FIVE-YEAR
PERIOD OF TIME.
$22 MILLION CAME FROM THE NIH.
WE ARE AUTHORIZED BY CONGRESS TO
RECEIVE AN ANNUAL TRANSFER OF
RESOURCES, BUT WE ALSO AS WE DID
WITH SOME OF THE COVID WORK,
RECEIVED A DIRECT TRANSFER OF
MONEY FROM NIH TO ACCELERATE OUR
ABILITY TO EXECUTE THE PORTFOLIO
OF COVID STUDIES.
AND NIH ALSO HAS SOME IMPORTANT
IN-KIND SUPPORT FOR OUR IT
SYSTEM SO ALL OF OUR DATA
SYSTEMS ARE CONCORDANT AND
COMPLIANT WITH DATA PRIVACY, ET
CETERA, THAT NIH REQUIRES.
THE REST OF OUR RESOURCES ARE
RAISED FROM OTHER SOURCES AND AS
YOU CAN SEE THE NIH IS REALLY A
MINORITY OF OUR INPUT.
WE RAISE A SIGNIFICANT AMOUNT OF
MONEY FROM LIFE SCIENCE
COMPANIES AND OTHER CORPORATE
ENTITIES, BUT ALSO A FAIR AMOUNT
FROM FOUNDATIONS.
THAT FOUNDATION SUMMIT IS
ANOTHER AREA THAT IS INCREASING
AS OUR INVESTMENT IN
PHILANTHROPIC INTERESTS.
WHERE DOES THE MONEY GO?
THE VAST MAJORITY OF OUR
RESOURCES TO GO FOR DIRECT
RESEARCH SUPPORT.
WE HAVE A NUMBER OF SCIENTISTS
INSIDE OF FNIH, A GROWING
NUMBER, WE HAVE DOUBLED IN SIZE
IN THE TWO YEARS I HAVE BEEN
WITH THE FOUNDATION AND THE VAST
MAJORITY ARE SCIENTIFIC PH.D.
TYPES.
OUR CHARITY NAVIGATOR RATING
REMAINS VERY HIGH.
WE ARE AN EFFICIENT
ORGANIZATION.
SO GETTING BACK TO THE
ACCELERATED MEDICINES
PARTNERSHIPS AND OUR
OPPORTUNITIES FOR INTERVENTION
ALONG CONTINUUM OF THE
BIOMEDICAL PIPELINE, WHICH I PUT
IN CARTOON FORM HERE.
WE HAVE ACTIVITIES BASICALLY IN
EVERY PHASE OF THIS THAT INVOLVE
PARTNERSHIP OR SUPPORT FOR
SPECIFIC SCIENTIST, BUT A MAJOR
CONTRIBUTION IS IN THE AREA OF
TARGET IDENTIFICATION.
BY TARGET AREAS WHERE WE ARE
IDENTIFYING IN OPPORTUNITIES FOR
DIAGNOSTIC OR THERAPEUTIC
DEVELOPMENT.
ON THE NEXT SLIDE, YOU CAN SEE
THE PROGRAM CALLED THE
ACCELERATING MEDICINES
PARTNERSHIP, A TRADEMARKED
ORGANIZATION BY THE NIH.
IT HAS BEEN IN EXISTENCE OVER 10
YEARS.
WE CELEBRATED THE 10th
ANNIVERSARY LAST SPRING.
THERE HAVE BEEN A TOTAL OF 10
PROJ
PROJECTS, TOTAL INVESTMENT OF
ALMOST $1 BILLION.
34 PARTNERS FROM LIFE SCIENCE
COMPANIES, 16 NIH INSTITUTES AND
CROSS INSTITUTE PROGRAMS AND A
NUMBER OF NONPROFIT
ORGANIZATIONS HAVE COME TOGETHER
TO PARTICIPATE IN THE PROJECTS
THAT ARE SHOWN ON A TIMELINE IN
THE NEXT SLIDE.
THE PROGRAMS REALLY STARTED WITH
ALZHEIMER'S DISEASE AND HAVE
CONTINUED NOW TO ENCOMPASS A
WHOLE RANGE OF THERAPEUTIC AREAS
INCLUDING PARKINSON'S, TYPE 2
DIABETES, HEART FAILURE, ALS, ET
CETERA, ET CETERA, OUTLINED HERE
IN THE TIMELINE.
ONE OF THE THINGS THAT MATTERS
TO ME IN THIS TIMELINE IS THAT
THE PARTNERS FIND THAT AT THE
END OF WHAT NORMALLY A FIVE-YEAR
CYCLE OF INVESTMENT IN A
PARTICULAR AMP AREA, OFTEN SAY,
NO, WE CAN GET MORE FROM THIS.
WE CAN DO MORE.
WE WANT TO LEARN MORE.
WE CAN CONTRIBUTE MORE.
LET'S GO TO THE 2.0 VERSION.
AND SO THEY ANTE UP AGAIN TO
PARTICIPATION AND PLAY.
-- PARTICIPATE AND PLAY.
I WANT TO EMPHASIZE ACROSS ALL
OF THESE PROJECTS LOOKING AT
IDENTIFYING NEW PATHWAYS AND
TARGETS FOR DRUG THERAPY DATA
ARE OPEN ACCESS.
THAT MEANS INFORMATION THAT IS
ACCRUED AND MANAGED THROUGH
THESE PROJECTS IS OPEN SOURCED
SO ANY RESPONSIBLE CREDENTIALED
INVESTIGATOR CAN USE THOSE DATA
FOR SCIENTIFIC PUMPS.
PURPOSES.
ON THE NEXT SLIDE I HAVE ONE
VIEW OF THE MEASUREMENT OF THE
IMPACT OF THESE PROJECTS ON
TARGET IDENTIFICATION AND
PRIORITIZATION ACROSS THREE OF
THE MAIN SECTORS WHO ARE
CONTRIBUTING.
WHAT THIS GRAPH SHOWS YOU IS
SOMETIMES THE AMP HELPS DEFINE
NEW TARGETS FOR EXPLORATION AND
DRUG DEVELOPMENT.
SOMETIMES THE AMPS MOVE SOME
PUNITIVE TARGETS HIGHER UP ON
THE PRIORITIZATION, THE MIDDLE
PART OF THE GRAPHIC FOR
ALZHEIMER'S, RHEUMATALOGIC AND
THEY CAN VERY QUICKLY LEARN
THOSE TARGETS DON'T MATTER AND
WE ARE NOT GOING TO PURSUE THOSE
ANYMORE AND TAKE THEM OFF THE
TABLE.
WHEN WE HAD OUR 10th YEAR
ANNIVERSARY OF THE AMPS, I SPOKE
TO THE INDUSTRY PARTNERS TO
REALLY UNDERSTAND WHY ARE YOU
CONTRIBUTES.
WHAT ARE YOU GETTING OUT OF
THIS?
ONE OF THE MOST IMPORTANT THINGS
THEY SAID IS WE WOULD NEVER BE
ABLE TO AMALGAMATE THE TYPE OF
INFORMATION IN THE
PRECOMPETITIVE ENVIRONMENT
SHARING DATA ACROSS VARIOUS
ORGANIZATIONS AND REALLY LOOKING
AT THE STATISTICAL POWER WE NEED
TO PRIORITIZE THESE TARGETS AND
MOVE THEM FORWARD.
IT IS WORTH EVERY PENNY THAT WE
INVEST TO TRY TO DO THAT AND
ACCELERATE OUR CAPABILITIES.
ON THE NEXT SLIDE I WANTED TO
JUST SAY A WORD ABOUT HOW WE ARE
EVOLVING THE AMPS BECAUSE THEY
ARE, AS YOU SAW IN THE TIMELINE,
FAIRLY LONG-TERM PROJECTS.
IT TAKES US A LONG TIME TO GET
THEM STARTED.
TRADITIONALLY 18 TO 24 MONTHS
FROM THE TIME WE HAD THE
APPROVAL TO MOVE AHEAD ON AN AMP
IN THE ACTUAL LAUNCH OF THE
INVESTMENTS IN THE SCIENTIFIC
PROCESS.
SO WHEN WE TOOK A LOOK AT WHY
THE TIMELINE WAS AS LONG AS IT
WAS, ONE OF THE REASONS IS THE
NIH BUDGET CYCLE HAS A LONG LEAD
TIME AND ISN'T ALWAYS KNOWN OR
ACCESSIBLE.
OUR PHILOSOPHY BEING A SMALL
FOUNDATION IS THAT WE DON'T WANT
TO DO THINGS AT RISK BECAUSE WE
CAN'T AFFORD TO MAKE OUR OWN
INVESTMENT AND THEN NOT HAVE
THAT END UP BEING SOMETHING THAT
IS FUNDED.
BUT WE ALSO FOUND OUT THAT THERE
ARE WAYS WE COULD DO THINGS IN
PARALLEL NOT IN SERIES AND MOST
RECENTLY AS IS EXEMPLIFIED BY
THE LAUNCH OF THE AMP ALS
CHANGING OUR CONTRACTING
MECHANISMS WITH THE PARTNERS, BY
DEVELOPING SOMETHING WE CALL THE
ACCELERATION FUND WHERE
PHILAN
PHILANTHROPISTS CONTRIBUTE TO
HIGH PRIORITY AND HIGH
PROBABILITY AT RISKS.
WE CAN SPEED UP THIS PROCESS.
WE ARE PROUD OF THE EXEMPLAR OF
THE ALS.
FOR THOSE OF YOU WHO KNOW PEOPLE
WHO ARE EXPERIENCING THE
CHALLENGES OF ALS, YOU KNOW THAT
EVERY DAY MATTERS AND SO
EVERYTHING WE CAN DO TO
ACCELERATE PROJECTS LIKE THIS
REALLY DOES MATTER TO THE
PATIENT STAKEHOLDERS WHO ARE
VERY MUCH PART OF OUR PROCESS.
I DO WANT TO SAY A WORD ABOUT
AMP DERIVATIVES AND ONE OF THE
AREAS WE HAVE MOVED FORWARD INTO
THE CLINICAL STUDIES ARENA.
LARRY IS HERE.
I MET MR. TABAK WHEN I STEPPED
UP TO MY ROLE AT FNIH AND STILL
PROSECUTING THE ACTIVE PORTFOLIO
OF IMPORTANT CLINICAL TRIALS
RELATIVE TO THE COVID, SORT OF
BY THE NUMBERS.
AND I THINK THAT IS THE SLIDE
YOU ARE SEEING IS SHOWING THAT
AC
ACTIVE CAME TOGETHER, DR.
COLLINS AND DR. WALLY LEAD THE
EFFORT TO SCREEN 800 COMPOUNDS,
ULTIMATELY LAND ON 33 COMPOUNDS
FOR EVALUATION.
SEVERAL OF WHICH PROVED TO BE
EFFECTIVE.
SEVERAL OF WHICH PROVED TO NOT
BE EFFECTIVE.
JUST INVOLVING MORE THAN 23,000
PATIENTS AT 620 CLINICAL SITES
AROUND THE UNITED STATES.
A MASSIVE UNDERTAKING AT RAPID
SKILL THAT THE NEXT SLIDE LISTS
THE MAIN PARTNERS PARTICIPATING
AND ACTIVE.
THIS EXPERIENCE IS VERY
IMPORTANT BECAUSE WE ALL LEARNED
FROM IT.
I THINK WE LEARNED SOMETHING
THAT WE'RE CARRYING FORWARD.
PARTICULARLY DR. BERTAGNOLLI'S
DIRECTION AND WE MOVE TO EXPAND
THE RECOVER, ON THE NEXT SLIDE,
THE RECOVER LONG COVID PROGRAM
TO ENGAGE MORE EFFICIENTLY AND
EFFECTIVELY IN THE DEVELOPMENT
OF THERAPEUTIC TRIALS FOR LONG
COVID.
SO IN VERY SHORT ORDER, I THINK
WE HAD ABOUT, MONICA, TWO OR
THREE-MONTH LEAD TIME HERE.
WE WERE ABLE TO CONVENE AS MANY
HUNDRED STAKEHOLDERS IN PERSON,
ON THE NIH CAMPUS, BUT ALSO
VIRTUALLY, WHICH INCLUDED A HUGE
CONSTITUENCY OF PEOPLE AFFECTED
BY LONG COVID AND THEIR CARE
TAKERS TO UNDERSTAND WHAT IS
NEEDED?
WHAT DO PATIENTS NEED?
WHAT ARE PATIENTS DOING NOW AND
HOW CAN WE ACCELERATE THE
CLINICAL TRIALS FOR THIS?
WE ARE MOVED INTO THE PHASE OF
SOLICITING POTENTIAL COMPOUNDS
FOR STUDY OR INTERVENTIONS FOR
STUDIES, NOT JUST ALL DRUG
RELATED INTERVENTIONS AND ARE
LEARNING FROM THE ACTIVE MODEL
AND WHAT WE'VE LEARNED FROM AMP
BUILDING COMPLEX PARTNERSHIPS TO
PROSECUTE THIS PORTFOLIO.
LET ME MOVE ON NOW.
YOU CAN SKIP THE NEXT SLIDE
WHICH I THINK JUST SAYS CLINICAL
STUDIES AND MOVE TO ANOTHER VERY
IMPORTANT EVOLUTION OF THE AMP
PROCESS WHICH IS THE CLINICAL
TRIALS RELATED TO LUNG MASTER
PROTOCOLS.
WHAT HAPPENS HERE AND YOU CAN
SEE HERE ON THE MAP, THE
GEOGRAPHIC DISTRIBUTION OF THESE
PROJECTS WHICH HAVE SO FAR
EVALUATED 15 INVESTIGATIONAL
DRUGS FOR CANCER.
RATHER THAN DOING 15 SEPARATE
TRIALS OR MORE THAN 15, THE
MASTER PROTOCOL ALLOWS PEOPLE TO
HAVE THEIR CANCERS INTERROGATED
FOR A GROWING LIST OF MARKERS
FOR SUSCEPTIBILITY TO VARIOUS
DRUGS AND THEN MODIFY THEIR
TREATMENT PROTOCOLS ACCORDING TO
WHAT WE LEARN ABOUT THOSE
MARKERS AND THE BENEFITS OR
NONBENEFITS OF THESE
INVESTIGATIONAL DRUGS.
THIS IS ANOTHER MODEL THAT HAS
BECOME VERY IMPORTANT IN
DEFINING MORE RAPID WAYS AND
MORE INCLUSIVE WAYS TO PROSECUTE
CANCER PORTFOLIOS, BUT ALSO IS
INFORMING OVER AND BROADER WORK
IN TEAM SCIENCE THAT WE ARE
CARRYING ON AS WE GO FORWARD.
WHICH BRINGS ME ON THE NEXT
SLIDE TO THE BIOMARKERS
CONSORTIUM PLATFORM.
WHICH IS NOT THE LARGEST
INVESTMENT THAT FNIH MAKES.
IN FACT, MOST OF THE INVESTMENT
IN THE BIOMARKERS CONSORTIUM IS
FROM THE PRIVATE SECTOR.
NIH CONTRIBUTES TO SOME OF THESE
35 PROJECTS THAT ARE ONGOING
HERE, BUT MOST OF THE RESOURCES
CONTRIBUTED BY THE OUTSIDE
PARTNERS.
WE'RE VERY INTENSE IN
PARTICIPATION OF THE ACADEMIC
COMMUNITY IN THIS REGARD AS
WELL.
THE PURPOSE OF THE BIOMARKERS
CONSORTIUM IS TO DEFINE BIOMARK
EARRINGS FOR VARIOUS DISEASES
THAT WOULD BE POTENTIALLY BE
VALIDATED AND THEN ULTIMATELY
QUALIFIED BY THE FDA TO SERVE AS
PREDICTORS, PROGNOSTICATORS OR
SURROGATE END POINTS FOR
CLINICAL TRIALS.
THIS IS A LENGTHY PROCESS.
THE ERA SINCE FDA CREATED
BIOMARKER QUALIFICATION, VERY
FEW HAVE MADE IT THROUGH THE
FUNNEL TO GET TO THAT POINT.
ON THE NEXT SLIDE I JUST SHOW
THE PARTNERS WHO PARTICIPATE IN
THE BIOMARKERS CON SSORCONSORTI.
I PUT THESE SLIDES UP FOR TWO
REASONS.
THIS IS A VERY DIVERSE
CONSTITUENCY OF PEOPLE WHO SHARE
ABOUT LEARNING ABOUT BIOMARKERS
ALL BOATS WILL FLOAT IN THE
RISING TIDE OF WHAT WE LEARN.
THE MODEL IS DIFFERENT THAN THE
AMP MODEL AND WE ARE THINKING
ABOUT HOW TO BRING THESE TWO
MODELS TOGETHER.
BECAUSE THE BIOMARKERS
CONSORTIUM IS A SUBSCRIPTION
MODEL.
PEOPLE PAY TO BE MEMBER OF THE
CONSORTIUM AND CAN OPT IN AND
OUT OF SPECIFIC PROJECTS AS THEY
DEVELOP AND EMERGE AND THAT IS
AN IMPORTANT MODEL AS WE THINK
ABOUT RESOURCE CONSTRAINTS.
ON THE NEXT SLIDE I HAVE A BIG
WIN FOR THE BIOMARKERS
CONSORTIUM, FIRST HINTED AT LAST
SPRING AT THE SCIENTIFIC MEETING
OF THE ASBMR.
BUT BASICALLY THE WORK FROM THE
BIOMARKER CONSORTIUM HAS
DEMONSTRATED THAT BONE MINERAL
DENSITY CAN BE USED AS A
SURROGATE END POINT FOR
DETERMINING THE EFFICACY OF
DRUGS FOR OSTEOPOROSIS.
THE FDA HAS VALIDATED THE
INFORMATION FROM THE INFORMATION
STUDIES AND CREATED A TIMELINE
THAT SAID AFTER COMPLETING
VARIOUS QUESTIONS AND ANSWERS
THAT NEED TO HAPPEN, WE EXPECT
WE WILL FULLY QUALIFY THIS
BIOMARKER BY JANUARY OF 2025.
WELL, GUESS WHAT?
JANUARY OF 2025 IS ALMOST HERE
AND SO WE'RE HOLDING OUR BREATH
THAT WE ACTUALLY WILL SEE THIS
QUALIFIED BIOMARKER.
WHY IS THIS IMPORTANT?
IT IS IMPORTANT BECAUSE
TRADITIONALLY IF YOU WANTED TO
STUDY A DRUG FOR OSTEOPOROSIS,
YOU HAD TO EN3R08,000 PEOPLE AND
FOLLOW THEM FOR 10 YEARS AND SEE
IF THEY DID OR DID NOT DEVELOP A
HIP FRACTURE.
THAT IS AN INCREDIBLY EXPENSIVE
UNDERTAKING AND VERY CAPITAL
TENSE AND TIME INTENSE, IT
DOESN'T HAPPEN VERY LONG THAT
PEOPLE INVEST THAT RESOURCE.
EN THOE OSTEOPOROSIS IS SO
IMPORTANT AND IMPROVING THE
QUALITY OF LIFE PARTICULARLY FOR
WOMEN AND PEOPLE WHO ARE AGING.
WITH A SURROGATE END POINT YOU
CAN SPEED UP THE TRIAL SO THE
END POINT ISN'T FRACTURE, THE
END POINT IS CHANGE IN BONE
MINERAL DENSITY.
YOU CAN DO THE STUDY WITH FAR
FEWER PEOPLE AND A STUDY THAT
MAY HAVE BEEN 10 YEARS COULD BE
SHORTENED TO SAY THREE YEARS.
WE REALLY HAVE AN OPPORTUNITY TO
GET PEOPLE BACK IN THE GAME OF
DEVELOPING NOVEL THERAPIES.
FOR OS TEOPOROS SIXTH.
IT SHOWS PEOPLE COMING TOGETHER
AND INTERROGATING PROBLEMS
TOGETHER CAN LEAD TO SIGNIFICANT
IMPACT.
THE LAST MAJOR PROGRAM I WANTED
TO TOUCH ON IS KIND OF AN
EXTENSION OF THE AMPS INTO AN
AREA THAT IS RELATED TO THE
OPPORTUNITIES FOR REGULATORY
APPROVAL OF GENE THERAPY.
I THINK I HIGHLIGHTED THAT ON
THE NEXT SLIDE THAT SAYS
REGULATORY APPROVAL IN RED INC.
THE SLIDE AFTER THAT KIND OF
PRESENTS A CARTOON, REALLY, OF
WHAT WE ARE THINKING ABOUT IN
TERMS OF APPROACHES TO
DEVELOPING PLATFORM TECHNOLOGIES
FOR VARIOUS KINDS OF GENE AND
CELL THERAPY.
ANYBODY WHO IS FAMILIAR WITH FLU
VACCINE KIND OF UNDERSTANDS THE
PLATFORM CONCEPT OF SLIPPING IN
AN ANTIGEN OR A MOLECULE IN A
BACKBONE THAT STAYS CONSTANT.
THAT IS A GREAT IDEA FOR CERTAIN
KINDS OF GENE AND CELL THERAPY.
WE ARE A LONG WAY FROM THAT
TODAY.
TOGETHER WITH NIH AND A VARIETY
OF PARTNERS FNIH IS LEADING AN
EFFORT TO TRY TO DEVELOP THE
PROCESSES OF MANUFACTURING THESE
KINDS OF PLATFORMS, BUT ALSO
IMPROVING CERTAINTY ABOUT THE
REGULATORY REQUIREMENTS FOR
GETTING THEM APPROVED.
SO NEXT SLIDE, THE ACCELERATING
MEDICINES PARTNERSHIP BESPOKE
GENE THERAPY CONSORTIUM WAS
CREATED.
THIS IS KIND OF A HYBRID BETWEEN
THE TRADITIONAL AMPS, THE
BIOMARKERS CONSORTIUM AND THE
CLINICAL TRIAL PLATFORMS I HAVE
BEEN TALKING ABOUT.
IT KIND OF BRINGS IT ALL
TOGETHER.
EARLY DAYS, BUT ON THE NEXT
SLIDE YOU CAN SEE A LITTLE BIT
OF WHERE WE ARE STARTING.
FIRST OF ALL, FROM THE
PARLIAMENT PER SP -- PATIENT
PERSPECTIVE, WE HAVE TO STOP
EXPECTING PEOPLE TO RAISE MONEY
THROUGH BAKE SALES AND MARATHONS
AND FUND THIS KIND OF RARE
DISEASE RESEARCH IN A SYSTEMATIC
WAY.
WE RECEIVED A GREAT DEAL OF
PATIENT INPUT AND SUPPORT FOR
THIS EFFORT.
THE FIRST EIGHT RARE DISEASES
THAT WE ARE APPROACHING WITH
CLINICAL TRIALS ARE ALL USING
AAB VECTORS.
THAT DOESN'T MEAN WE ARE LIMITED
TO THAT.
WE'VE ALREADY BEEN EXPLORING
BEYOND AAV VECTORS.
WE HAD TO START SOMEWHERE.
THROUGH A COMPLEX PROCESS THAT
WAS KIND OF BASED ON WHAT WE
LEARNED FROM ACTIVE, WE
PRIORITIZED THE TARGETS AND THE
MOLECULES THAT ARE THE FIRST
PASS AT THE AREAS THAT WE'RE
OPERATING FROM.
NEXT SLIDE SHOWS THE INCREDIBLE
NIH ENGAGEMENT ACROSS 10
INSTITUTES, FDA OBVIOUSLY A HUGE
COMPONENT OF THIS PARTICULAR
PARTNERSHIP AND A VARIETY OF
EXTERNAL PARTNERS FROM LARGE AND
SMALL COMPANIES, PHILANTHROPY,
PATIENT ORGANIZATIONS, ET
CETERA.
THIS IS NO LONGER A COMPLETE
LIST OF PARTNERS.
SOME OF THE PATIENT
ORGANIZATIONS PARTICIPATING ARE
NOT THE PATIENT ORGANIZATIONS
WHOSE DISEASES WERE REPRESENTED
ON THE PREVIOUS SLIDE, BUT THEY
ARE IN BECAUSE THEY RECOGNIZE
THIS IS REALLY IMPORTANT TO
OVERALL PROGRESS IN THE FIELD OF
CELL AND GENE RESEARCH.
OUR FIRST CONTRIBUTION ON THE
NEXT SLIDE WAS RELEASED IN
FEBRUARY, THE FIRST PLAYBOOK TO
DEFINAL SOME OF THE REGULATORY
STANDARDS FOR EVOLVING A
FRAMEWORK OF DEVELOPING VECTORS.
THE MINIMUM CQAs AND SOME OF THE
TOXICOLOGIC TO BEGIN THE
STREAMLINE OF STREAMLINING AND
REGULARIZING THE REGULATORY
APPROVAL, IF YOU WILL.
WE ARE EXCITED ABOUT HOW THIS
WORK COMES TOGETHER.
WE ARE EXCITED ABOUT THE FACT
THAT SCIENCE IS DEFINITELY ON
OUR SIDE THESE DAYS.
WHAT WE'LL BE ABLE TO DO
SCIENTIFICALLY IS TREMENDOUS.
WE ARE ALL OPERATING IN THE
SPIRIT OF CONSTRAINED RESOURCES
AND HAVE TO LEARN TO DO MORE,
BETTER, FASTER, WITHOUT
EXPECTING THE BUDGET TO BE THE
SOLUTION TO THAT.
I THINK THERE IS A ROLE FOR
PARTNERSHIPS AND I HOPE FNIH
WILL BE CONTINUING TO SUPPORT
THE NIH IN ITS MISSION AS WE GO
FORWARD.
THE NEXT SLIDE KIND OF REMINDS
YOU OF THE VARIOUS THINGS WE DO
AND VARIOUS FORMATS AT FNIH TO
BRING THIS ALL TOGETHER.
I THINK MAYBE IN THE INTEREST OF
TIME I'LL STOP HERE SO WE HAVE
TIME FOR QUESTIONS AND ANSWERS.
I DO WANT TO SAY ONE OTHER
THING.
IF I CAN JUMP AHEAD TWO SLIDES
TO THE SLIDE THAT SAYS
SUPPORTING SCIENTISTS.
BECAUSE I REALIZE WHEN I WAS
LOOKING AT THE ADVISORY
COMMITTEE TO THE DIRECTOR THAT
DR. KANG WAS A MEMBER OF THE
ADVISORY COMMITTEE TO THE
DIRECTOR.
WE JUST OFFERED DR. CHANG AS THE
LURIE PRIZE WINNER FOR HIS
INCREDIBLE SCIENTIFIC
CONTRIBUTIONS ALONG WITH OTHER
SCIENTISTS WHO ARE HONORED NOW
IN THIS ENDOWED PRIZE CALLED THE
PAUL GOWAN TRAILBLAZER PRIZE
NAMED AFTER DR. GOWAN AND
PARTNERSHIP AWARDS MENTIONED
HERE.
IT GIVES ME A CHANCE TO SAY AS I
TRIED TO SAY AT THE BEGINNING,
TEAM SCIENCE IS THE CORE OF OUR
WORK, WE DO A LOT OF WORK TO
SUPPORT INTRAMURAL SCIENTISTS IN
THE LAB AT NIH WHERE WE CAN
CHANNEL PHILANTHROPISTES AND
TO -- MOST RECENTLY THE SAWYER
LECTURE AND THE EYE INSTITUTE IS
AN EXAMPLE OF HOW WE TRY TO
BRING SCIENCE TO THE FOREFRONT
AND HONOR THE LEGACY GIVING OF
SOME OF THE IMPORTANT
STAKEHOLDERS.
ON THE NEXT SLIDE BECAUSE TRUST
IN SCIENCE IS SO IMPORTANT, I'M
SURE IT IS IMPORTANT TO THE ACD,
WE ARE TRYING TO STEP UP TO
RECOGNIZING OUR ROLE AND THIS
YEAR DEVELOPED A NEW PRIZE AT
OUR ANNUAL AWARDS DINNER FOR
TRUST IN LIFE SCIENCE
JOURNALISM.
THE PERSON WHO WON THE PRIZE WAS
KATHERINE WU, A STAFF WRITER FOR
THE ATLANTIC MONTHLY, BUT
PARTICIPATED IN CREDIBLE NEWS
REPORTING AT THE "NEW YORK
TIMES" AND VARIOUS OTHER MEDIA
OUTLETS AS A BALANCED AND FAIR
SCIENCE REPORTER.
WE HAVE BEEN PARTICIPATING FOR
THE COALITION FOR TRUST AND
HEALTH IN SCIENCE, NOT TO TELL
PEOPLE WHAT IS TRUTH AND NOT
TRUTH RATHER TO HELP PEOPLE
UNDERSTAND WHERE THEY CAN GO TO
GET RELIABLE INFORMATION TO HAVE
WHAT THEY NEED TO MAKE THE
CHOICES THAT ARE IN FRONT OF
THEM.
LET ME STOP THERE.
AGAIN, REMINDING YOU OF OUR
AMBITION AND OUR INTENT, BUT
ALSO THANKING YOU ALL FOR YOUR
PARTNERSHIP, PARTICULARLY THE
LEADERS OF THE NIH, INCLUDING
DR. HODES WHO IS FOLLOWING ME,
WHO HAVE BEEN INCREDIBLE
PARTNERS WITH THE FNIH AND I
HAVE LEARNED SO MUCH AND FEEL
VERY GRATEFUL FOR MY OPPORTUNITY
TO PARTICIPATE WITH ALL OF YOU.
SO THANK YOU.
>> OH, JULIE, THANK YOU SO MUCH.
I JUST WANT TO SAY MY SLOGAN FOR
NIH IS THAT OUR JOB IS NOT DONE
WHEN WE DELIVER SCIENTIFIC
DISCOVERIES.
OUR WORK IS ONLY DONE WHEN
PEOPLE ARE LIVING LONG AND
HEALTHY LIVES.
THE FOUNDATION FOR THE NIH IS
OUR MAIN PARTNER IN BEING ABLE
TO DO THAT, TO BRIDGE THAT GAP
BETWEEN WHAT SCIENCE CAN DELIVER
AND THEN BUILD THE PARTNERSHIPS
AND I LOVE THE WAY YOU SAY IT,
PUBLIC-PRIVATE-PERSON
PARTNERSHIPS.
I'M GOING TO USE THAT FOREVER.
AND TO REALLY CHANGE HEALTH AND
DELIVER OUTCOMES THAT MATTER TO
PEOPLE.
YOU'RE AN AMAZING PARTNER.
IT IS AN INCREDIBLE ORGANIZATION
AND WE ARE SO GRATEFUL FOR
EVERYTHING FNIH DOES.
NOW WE CAN TAKE SOME QUESTIONS
FOR YOU.
>> OKAY.
SO LET'S SEE WHO WANTS TO GO
FIRST, PLEASE.
YES.
RUSS.
>> SO DR. GERBERDING, FABULOUS
PRESENTATION AND A REMARKABLE
ORGANIZATION.
I KNOW IN OUR WORLD THE
RETINITIS PIGMENT DPLS TOS TRIAL
AND THE REGULATION CAN SLOW
THESE THINGS AND IT TAKES A
CONSORTIUM TO PUSH FORWARD IN A
LOGICAL AND REASONABLE WAY.
I HAD A QUICK PEEK AT YOUR 990s
DURING THE PRESENTATION.
I GUESS THE QUESTION I HAVE FOR
YOU AS AN UNUSUAL
CONGRESSIONALLY CHARTERED 501C3,
SUSTAINABILITY IS A CHALLENGE.
YOU HAVE A MODERATE CORPUS,
ABOUT $200 MILLION AND ABOUT A
$60 MILLION A YEAR BUDGET, WHICH
IS REMARKABLE BECAUSE THE AMOUNT
OF WORK COMING OUT LOOKS LIKE AN
INSTITUTE OR CENTER OUTPUT ON A
RELATIVELY SMALL BUDGET.
WHAT'S THE LONG-TERM PLAN FOR
SUSTAINABILITY AND CAN YOU TALK
A LITTLE BIT ABOUT HOW SUCCESSES
MAY FEED BACK FINANCIALLY, FOR
EXAMPLE, WHEN THERE ARE LICENSES
AND ROYALTIES AND THINGS OF THAT
SORT AND WHAT IT IS GOING TO
TAKE TO KEEP THIS VERY VALUABLE
ORGANIZATION SUSTAINED?
>> THANK YOU SO MUCH FOR THAT.
AND THERE'S A LOT IN YOUR
QUESTION.
SO LET ME FIRST SAY THAT ONE
THING FOR THOSE WHO MIGHT NOW BE
LOOKING AT OUR 990s, IN SOME
COLUMNS IT LOOKS LIKE WE HAVE
MILLIONS OF DOLLARS.
BUT ONE FACT IS WHEN WE START A
PROJECT AND WE BUILD THE
RESERVOIR OF MONEY TO PAY FOR
IT, WE GATHER ALL THE MONEY
BEFORE THE PROJECT BEGINS SO WE
SIT ON A LARGE ROLLING FUND OF
MONEY THAT ULTIMATELY GOES OUT
TO SUPPORT THOSE INVESTMENTS
THAT WE MAKE IN ACADEMIA AND IN
OUR OTHER PARTNERS.
SO THERE IS KIND OF A FALSE
SENSE THAT WE HAVE A LARGESS
WHEN, IN FACT, WE DO NOT AS YOU
HAVE PROPERLY POINTED OUT.
WE HAVE AN EXTREMELY SMALL
ENDOWMENT FOR AN ORGANIZATION OF
OUR SIZE.
ONE OF THE FIRST THINGS I
REALIZED WHEN I STARTED IS WE
NEED TO WORK ON BUILDING OUT OUR
ENDOWMENT SO WE HAVE MORE
SUSTAINABLE OUTLOOK AND THAT
MEANS WE'RE ASKING TO DO THAT AT
A TIME WHEN WE KNOW WE'RE NOT IN
THE SAME BOAT WE WERE IN 10
YEARS AGO WHEN THE LIFE SCIENCE
INDUSTRY HAD MONEY TO SPARE.
THEY ARE ALSO EXPERIENCING
CONSTRAINTS ON THEIR RESOURCES.
SO WE CAN'T KEEP GOING TO THAT
WELL AND EXPECTING THEM TO FUND
OUR ENDOWMENT.
WE HAVE TO RAISE THAT MONEY.
AND THAT MEANS PHILANTHROPY.
WE ARE OPERATING IN A WORLD
WHERE THERE ARE INCREASING
DOLLARS AVAILABLE FOR
PHILANTHROPY.
WE HAVE TO SHOW THE VALUE OF
THAT INVESTMENT, THAT WE ARE
APPEALING TO PEOPLE WHO REALLY
CARE ABOUT MEDICAL RESEARCH IN
SPECIFIC THERAPEUTIC AREAS AND
PEOPLE WHO UNDERSTAND THE LONG
GAME OF SCIENCE.
HAVING SAID THAT, WE HAVE A
GREAT STORY TO TELL BECAUSE WHO
ELSE BRINGS TOGETHER THE
SMARTEST PEOPLE AT THE NIH WITH
THE SMARTEST PEOPLE IN INDUSTRY
AND IN INDUSTRY AND THE PATIENT
COMMUNITY TOGETHER?
THE PROBABILITY OF SUCCESS
MATTERS AND WE HAVE CREATED A
KOR -- CONSORTIUM OF PEOPLE.
IT IS FOR PEOPLE WHO ARE
SOPHISTICATED BIOSCIENCE WHY
THIS WOULD BE A WONDERFUL
CONTRIBUTION TO MAKE TO OUR
BIOMEDICAL RESEARCH ENTERPRISE.
ONE PARTICULAR AREA OF APPEAL
I'M WORKING ON FRAMING IS THE
IMPORTANCE OF SUPPORTING
EMERGING SCIENTISTS, HENCE THE
PRIZES I MENTIONED EARLIER, BUT
OUR SCIENTISTS ARE STRUGGLING IN
ACADEMIA FOR SUPPORT,
PARTICULARLY POST DOCS AND THE
MORE JUNIOR SCIENTISTS WORKING
THEIR WAY UP THE ACADEMIC
LADDER.
WE REALLY WANT TO BE ABLE TO
BRING THEM INTO THE FOLD OF TEAM
SCIENCE AND HELP THEM GET
LAUNCHED IN THEIR CAREER.
SO MUCH TO SAY ABOUT THE LONG --
I WOULD LOVE TO TALK WITH YOU
OFFLINE IF YOU ARE INTERESTED
BECAUSE WE ARE TRYING TO THINK
THROUGH THIS.
ONE OTHER QUICK THING ABOUT THE
VALUE OF THE IP OR THE
INTELLECTUAL PROPERTY THAT WE
MAY CREATE OR BE PRIVY TO, WE
ARE EXPLORING AND HAVE BOARD
APPROVAL FROM FNIH TO EXPLORE
THE FEASIBILITY OF A VENTURE
FUND.
MANY NONPROFIT ORGANIZATIONS
HAVE CREATED VENTURE FUNDS THAT
ARE EITHER BASED ON VENTURE
PHILANTHROPY OR TRUE VENTURE.
IS THERE INTELLECTUAL PROPERTY
NOT BEING EXPOSED.
EARLY DAS.
NOT SURE HOW IT WILL PLAY OUT
BUT WE ARE EXPLORES THAT
OPPORTUNITY.
>> THANK YOU.
MARK, YOU ARE NEXT.
>> HI, EVERYONE.
I'M NEW TO THE COUNCIL AND I WAS
TRAVELING ALL DAY YESTERDAY FROM
DENMARK.
I'M AT THE UNIVERSITY OF
VERMONT.
I LOVE THE INITIATIVE HERE.
I WAS JUST GOING TO SUGGEST
SOMETHING BASED ON WHAT WE DO.
WE HAVE SUPPORT FROM THE LADUKE
FOUNDATION FOR CARDIOVASCULAR
AND NEURAL VASCULAR OF
EXCELLENCE.
THEY HAVE A BROAD VIEW IN
CAMBRIDGE, MASSACHUSETTS.
THEY FUND ABOUT FOURNET NETWORKO
THE TUNE OF $8 MILLION A YEAR
FOR EACH NETWORK.
HAVING TALKED TO THEM AND HAVING
DEVELOPED SOME THINGS, THEY HAVE
AN ASTUTE VENTURE CAPITAL ARM
AND IT IS FOCUSED, WHAT WE ARE
INVOLVED IN ARE RARE MONOGENIC
CAUSES OF SMALL DISEASE OF THE
BRAIN WITH THE IDEA THIS WOULD
HAVE IMPLICATIONS FOR GREATER
DEMENTIA AND THEIR VIEW IS THE
RARE DISEASE, YOU ARE ALREADY
DOING THIS.
THE RARE DISEASES GIVE YOU A
WINDOW INTO THE MORE COMMON
ONES.
THEY ARE REALLY INTERESTED IN
DEVELOPING DRUGS AND THERAPIES
FOR THE RARE ONES WITH THE HOPE
THAT THEY WILL BE ABLE TO SPREAD
IT OUT TO A BROADER DISEASE
PROFILE.
SO ONE OF THE MAJOR ONES CALLED
CADACIL, A MONOGENETIC, IT IS
AUTOSOMAL DOMINANT.
IT IS YOUNG PEOPLE AND YOU CAN
IDENTIFY THEM.
BUT AGAIN IT IS PROBABLY THE
GENE MUTATION IS PROBABLY MORE
COMMON WITH PEOPLE WITH DEMENTIA
AND SMALL VESSEL DISEASE.
I WOULD SUGGEST OPENING IT UP TO
BRAIN DISEASES TO HIT DEMENTIA.
GO FOR THE GENETIC ONES FIRST
WITH THE IDEA YOU COULD LOOK AT
A BROADER SPECTRUM OF THE
DISORDERS THAT CAUSE.
I GOT A COLD ON THE PLANE.
NO COVID.
>> THANK YOU FOR THE LEAD.
I'M NOT FAMILIAR WITH BROAD VIEW
AND THIS SOUNDS LIKE SOMETHING
THAT COULD BE AN IMPORTANT
CONVERSATION.
THANK YOU.
>> HOOK UP THE LEDUCQ
FOUNDATION.
THEY ARE PROBABLY WORTH $1
BILLION.
THE BEAUTY OF THAT IS THEY ARE
BIG ON YOUNG INVESTIGATORS, BUT
NOT JUST YOUNG INVESTIGATORS IN
THE UNITED STATES AND NORTH
AMERICA, BUT IN EUROPE, TOO.
WHEN PEOPLE IN MY LAB GET TO
MAKE CONTACTS AND COLLABORATE
WITH PEOPLE IN GERMANY, FRANCE
AND SWEDEN.
THAT BROADENS THE RICHNESS OF
THE SCIENCE FOR THE YOUNG
INVESTIGATORS.
>> ABSOLUTELY.
THANK YOU.
>> THANK YOU SO MUCH.
OTHER COMMENTS OR QUESTIONS?
WE ARE GETTING NEAR THE TIME WE
HAVE TO ALLOW DR. GERBERDING TO
GET ON THE PLANE SHE IS GOING TO
BE CATCHING SHORTLY.
OKAY.
I'M NOT SEEING ANYBODY ELSE.
JULIE, THANK YOU SO MUCH.
MONICA, BACK TO YOU.
>> YEAH, THANKS, JULIE.
SAFE TRAVELS.
>> THANK YOU.
BYE-BYE.
THANK YOU.
>> WE ARE GOING TO HAVE ANOTHER
UPDATE AND DR. NELSON I THINK
YOU MIGHT BE INTERESTED IN THIS.
THIS IS AN UPDATE FROM
ACCELERATING MEDICINES.
ON THE ALZHEIMER'S DISEASE, AMP
FOR ALZHEIMER'S.
I AM GOING TO TURN IT OVER TO
DR. RICHARD HODES, THE NATIONAL
INSTITUTE OF AGING TO PRESENT
THIS AMP.
>> THANK YOU VERY MUCH FOR THE
CHANCE TO BE HERE.
IT IS GREAT TO BE FOLLOWING
JULIE.
I WOULD LIKE TO TAKE THIS TIME
TO RELATE TO YOU THE PROGRESS
THAT HAS BEEN MADE IN
ALZHEIMER'S DISEASE WHERE AMPS
ARE A VERY IMPORTANT PART OF
THAT.
JUST BY A BIT OF HISTORY, AN
IMPORTANT JUNCTURE IN
ALZHEIMER'S DISEASE RESEARCH
CAME WITH PASSAGE OF A NATIONAL
PLAN AND PROJECT ACT FIRST IN
2011 AND RE-AUTHORIZED WHICH
CALLED FOR A NATIONAL PLAN WITH
NIH PLAYING THE LEAD ROLE IN THE
RESEARCH PART OF THAT, RISK
REDUCTION TREATMENT, CARE,
SUPPORT AND PUBLIC AWARENESS.
DRAMATICALLY THIS IS TRANSLATED
INTO THE INCREASE OF NIH
SPENDING OR FUNDING FOR
ALZHEIMER'S AND RELATED
DEMENTIAS IN THE YEARS FROM 2015
TO 2023.
THAT IS THE LAST YEAR WE HAVE
COMPLETE DATA DRAMATIC SIX FOLD
INCREASE IN THAT FUNDING.
IT IS IMPORTANT IT REFLECTS THE
IMPORTANCE PLACED BY THE PUBLIC
IN A VERY BIPARTISAN WAY IN
RESEARCH IN THIS AREA.
IT CREATES AN IMPORTANCE
RESPONSIBILITY TO USE THESE
RESOURCES TOWARD INTENDED AIMS
IN PURSUIT OF TREATMENT OF THIS
DEVASTATING DISEASE.
FORTUNATELY, THE RESEARCH
OPPORTUNITIES AND SCIENCE HAVE
REGRESSED AT A PACE WHICH IS
WELL POSITIONED TO TAKE
ADVANTAGE OF THESE RESOURCES.
THIS IS GOING TO BE THE THEME OF
A LOT OF WHAT I WILL TALK ABOUT.
WHERE WE HAVE COME FROM 2015,
WHAT WE HAVE BEEN ABLE TO AND TO
THE PRESENT TIME.
ONE OF THE COMMITMENTS AS WE'VE
UTILIZED THE INCREASE FUNDING
FOR ALZHEIMER'S DISEASE RESEARCH
HAS BEEN TO MAKE SURE WE TOOK
ADVANTAGE OF ALL PERSPECTIVES
AND OPPORTUNITIES.
SO ALTHOUGH THE APPROPRIATIONS
HAVE LARGELY COME THROUGH THE
NATIONAL INSTITUTE ON AGING AND
NINDS, FROM THE BEGINNING WE
USED THOSE FUNDS TO SUPPORT
RESEARCH WHEREVER BEST CAN BE
DONE.
THIS IS AN EXAMPLE OF ONE OF
THOSE PROGRAMS.
GIVING ADMINISTRATIVE
SUPPLEMENTS TO GRANTS WHICH ARE
NOT TARGETING ALZHEIMER'S
RELATED DEMENTIA, BUT THE
SUPPLEMENT ALLOWS INVESTIGATORS
WITH GREAT TALENTS, ABILITIES
AND INSIGHTS IN OTHER AREAS TO
MOVE INTO THIS FIELD.
THE YEAR 2023, THE NUMBER OF
SUPPLEMENTS THAT WERE GIVEN,
NIA, 35, 35 OF THE 204
ESSENTIALLY ALL OF THE
INSTITUTES AND CENTERS, THE
GENIUS OF THOSE INVESTIGATORS
HAVE BEEN BROUGHT INTO THE FOLD
TO JOIN THE EFFORTS TARGETING
ALZHEIMER'S RELATED DEMENTIAS.
SO ALSO IN TERMS OF BEING
RESPONSIBLE.
THIS INCREASING SET OF
RESOURCES.
WE'VE EMBARKED ON A VERY
EXPLICIT AND TRANSPARENT FORMULA
FOR GENERATING OUR PRIORITIES.
THEY BEGIN WITH THE USUAL SETS
OF CONTACTS WITH MULTIPLE
MEETINGS.
IN THIS CASE WITH ANNUAL SUMMITS
AS WELL.
NATIONAL ACADEMIES REPORTS.
ALL OF THIS HAS GONE INTO THE
FORMATION OF A LIST OF
MILESTONES WHICH LAY OUT THE
SPECIFIC ACCOMPLISHMENTS NEEDED
TO PURSUE THE GOALS,
OPPORTUNITIES, THAT HAVE COME AS
A RESULT OF THE WIDE BROAD
INCLUSIVE AND TRANSPARENT INPUTS
FROM RESEARCH COMMUNITIES
ADVOCATES AND THOSE LIVING WITH
ALZHEIMER'S RELATED DEMENTIAS.
THE RESEARCH IMPLEMENTATION
MILESTONES ARE IN BROAD
CATEGORIES THAT FOLLOW TO THE
GOALS SET IN THE PLAN,
EPIDEMIOLOGY, DISEASE
MECHANISMS, DIAGNOSIS,
TRANSLATIONAL AND CLINICAL
INTERVENTIONS.
DEMENTIA CARE, EQUALLY IMPORTANT
FOR THOSE LIVING WITH DEMENTIA,
RESEARCH RESOURCES AND THERE ARE
TWO REPORTS I NOTE HERE FOR YOUR
INTEREST, ONE IS THAT EACH YEAR
AS A PART OF THE NATIONAL PLAN
AND CONGRESSIONAL REQUIREMENT,
NIH, THE NIH DIRECTOR PUTS
FARTHER A BUDGET OUTLINING TO
ACCOMPLISH THE GOALS OF THE
PLAN.
THE OTHER THING WE DO EACH-YEAR
IS PROVIDE A PROGRESS REPORT
ILLUSTRATING AND SUMMARIZING
PROGRESS IN THESE AREAS EACH
YEAR.
A LOOK AT THE INFRASTRUCTURE TO
BUILD RESEARCH OPPORTUNITIES
WITH THE MATURATION OF SCIENCE
AND RESOURCES THAT HAVE BECOME
AVAILABLE.
AROUND A FRAMEWORK, SO THE
DETERMINANTS FOR THE ALZHEIMER'S
AND RELATED DEMENTIAS CLEARLY
GENE ENVIRONMENT INTERACTIONS
THAT WE ARE USED TO, BUT ALSO
MAKE A NOTE OF AGING AS A
VARIABLE.
CLEARLY AGING IS THE MOST
IMPORTANT RISK FACTOR FOR
ALZHEIMER'S RELATED DEMENTIAS
AND THE NORMATIVE PROCESSES OF
AGING HAVE BECOME AN IMPORTANT
COMPONENT OF THESE OVERLAPPING
THEN DETERMINE NAPTS OF WHAT
ULTIMATELY LEADS YOU TO
PRECLINICAL AND CLINICAL
MANIFESTATIONS OF ALZHEIMER'S
AND RELATED DEMENTIAS.
SO INFRASTRUCTURE IS IMPORTANT.
JUST AS WE HAVE BEEN
EXPERIENCING THE INPUT FROM
INDIVIDUAL INVESTIGATORS, A LOT
OF WHAT YOU HAVE HEARD FROM
JULIE IN THE LAST CONVERSATION
HAS BEEN RELEVANT HERE.
TRYING TO FACILITATE THE
FORMULATION OF MEANS, TEAMS AND
AS SEAMLESS WAY AS POSSIBLE TO
PROVIDE A PIPELINE FOR
TRANSLATION FOR BASIC DISCOVERY.
BEGINNING WITH EARLY TARGET
IDENTIFICATION.
THE EARLY DAYS OF AMP THROUGH
DEVELOPING ASSAYS SCREENING FOR
THE TARGETS VALID PROOF OF
CONCEPT AND PRECLINICAL AND
CLINICAL TRIALS.
WE HAVE BEEN ABLE TO ESTABLISH
CENTERS BUILT ON A PARADIGM OF
OPEN SCIENCE, CONTRIBUTIONS BY
MULTIPLE GROUPS TO COMMON DATA
BASES, PORTALS WHICH ALLOW THE
UTILIZATION OF THESE DATA AND
OPTIMIZED APPLICATION TO THE R
IMPORTANT GOALS AND PROJECTS AT
HAND.
AS AN ILLUSTRATION HERE, THE
SYSTEMS BASED APPROACH TO
DECONSTRUCTING DISEASE.
ALZHEIMER'S ORIGINALLY DEFINED
BY PLAQUES AND TANGLES IS
CLEARLY IN THE CLINICAL
MANIFESTATIONS A FAR MORE
COMPLEX DISEASE IN UNDERLYING
PATHOLOGIES AND PROCESSES.
AMP AD, WE WILL TALK ABOUT IN
MORE DETAIL, EQUAL ENTERPRISES
TO LOOK AT THE VASCULAR DISEASE
AND RESILIENCE AND
NEUROPSYCHOSYMPTOMS.
SMALL IN THE MIDDLE THE AD
KNOWLEDGE PORTAL, EMBLEMATIC OF
HOW WE HAVE BUILT A COMMON
PORTAL TO RESOURCES TO POSITIVE
DATA MAKING IT AVAILABLE TO THE
ENTIRE RESEARCH COMMUNITY.
I WANT TO POINT OUT HERE THE
VERY IMPORTANT EVOLUTION, I
THINK WE'VE SEEN IN THE
SOCIALIZATION AND ATTITUDES OF
INVESTIGATORS.
MOVING FROM AS JULIE REFERRED TO
AS WELL THE SINGLE LAB AIMED AT
GETTING ITS RESEARCH PUBLISHED
TO A DRAMATIC AND RAPID
EVOLUTION OF THE STAGE OF
APPRECIATING THE ADVANTAGE THAT
COMES FROM POOLING DATA AND
MAXIMIZING THAT.
AMP AD GOT TO THE POINT WHERE
THE MAJORITY OF TRACK IMPORTANT
ADVANCES ARE PUBLISHED BY
INVESTIGATORS THEMSELVES AND NOT
GENERATE THE DATA.
THAT IS A GOOD AND IMPORTANT
HEALTHY SIGN OF THE DIRECTION WE
ARE TAKING.
SO TALK SPECIFICALLY ABOUT AMP.
AMP IS ONE OF THE ORIGINAL AMPLE
AD FOR ALZHEIMER'S DISEASE WAS
ONE OF THE INITIAL THREE OF THE
AMPS.
IT HAS BEEN A VERY EFFECTIVE
PUBLIC-PRIVATE PARTNERSHIP
BRINGING TOGETHER MULTIPLE
INSTITUTES AT NIH AND OTHER
GOVERNMENT AGENCIES SUCH AS FDA,
PHARMACEUTICAL, NONPROFIT
ORGANIZATIONS.
GENERATING A VERY CENTRALIZED
DATA RESOURCE OF HIGH QUALITY
RESEARCH APPLIED IN A DIRECTIVE
AND INTENSAL WAY.
FOR EXAMPLE, TEAMS OF
REPRESENTATIVES FROM THE PHARMA,
WHO HAVE BEEN A PART OF THE
ENTERPRISE ALONGS WITH LEADING
ACADEMIC INSTITUTIONS HAVE
IDENTIFIED POTENTIAL TARGETS,
OVER 1,000 AND TOOK THAT
OVERWHELMING NUMBER, IDENTIFIED
MOST PROMISING WITH TARGETED
RELATIONSHIP DISEASE, THOSE MOST
TARGETABLE IN PHARMALOGICAL
ENTERPRISE.
THIS IS NEW DISEASE SPECIFIC
INTERVENTIONS CAUSED PHARMA TO
MOVE IN NEW DIRECTIONS AND DROP
SOME OLD DIRECTIONS THAT WERE
NOT PROVING TO BE IMPORTANT OR
PRODUCTIVE.
AMP AD2.0 BE GINNING IN THE
SECOND ITERATION.
TEAMS FROM 20 ACADEMIC
INSTITUTIONS, NINE
PUBLIC-PRIVATE INSTITUTIONS
LOOKING TOWARD PRECISION
MEDICINE.
HERE THE BUZZ WORDS RELY IN THE
CASE OF ALZHEIMER'S RELATED
DEMENTIAS TO WHAT I MENTIONED.
WITHIN THE BRAINS OF INDIVIDUALS
WITH THIS CLINICAL SYNDROME
MULTIPLE PATHOLOGIES ARE
OCCURRING AND IT WILL BE
IMPORTANT TO TAILOR WHAT IS
HAPPENING IN EACH INDIVIDUAL IN
ORDER TO MAXIMIZE THE SUCCESS OF
THESE INTERVENTIONS.
GENETICS, THE ALZHEIMER'S
DISEASE SEQUENCES PROGRAM
STARTED VERY EARLY.
IT HAS MOVED TO THE POINT IN THE
LAST YEARS THE EMPHASIS IS VERY
PRODUCTIVELY TO INCREASE THE
GENETIC DIVERSITY OF THE
POPULATIONS THAT WE'RE STUDYING
AND BY LOOKING AT DIVERSE
POPULATIONS INCREASING NUMBERS
OF ASIAN, AFRICAN-AMERICAN,
HISPANICS, NONHISPANIC WHITES
WITH GLOBAL OUTREACH TO IDENTIFY
IMPORTANT INSIGHTS NOT JUST FOR
THE SUBJECT POPULATIONS BUT
OVERALL UNDERSTANDING OF THE
HET
HETER
HETEROG NEXT EI DWRRKS OF THE
DISEASE.
THE ALZHEIMER'S DISEASE RESEARCH
CENTERS NETWORK WE SUPPORTED IN
GREAT DIVERSITY OF THE NATION,
DIVERSITY IN TERMS OF
POPULATIONS.
MOST OF THE CENTERS HAVE
IMPORTANT ASPECTS OF OUTREACH TO
PRIMARY COMMUNITIES WHERE IT IS
IMPORTANT THE RESEARCH BOTH
RECRUITMENT OCCUR AND WHERE THE
INTERACTIONS WITH INDIVIDUALS
WHO ARE AT RISK FOR AND AFFECTED
WITH DEMENTIAS OCCUR IN DIVERSE
POPULATIONS ACROSS THE NATION.
IN ADDITION TO THE TARGETED
RESEARCH AIMED AT THE BIOLOGICAL
UNDERPINNINGS OF ALZHEIMER'S
DISEASE, WE HAVE RELIED
IMPORTANTLY AND WITH GREAT
SUCCESS ON LONGITUDINAL STUDIES
START WITH THIS INITIAL NATIONAL
POPULATION AND SHOW HOW IT HAS
BECOME GLOBALIZED.
THE HEALTH AND RETIREMENT STUDY
INITIATED BY HRS WANTED TO LOOK
AT WHAT HAPPENS AROUND THE
PERIOD OF LIFE AROUND
RETIREMENT, INDIVIDUALS 50 AND
OLDER.
IT WAS VERY MUCH IN THE
BEGINNING AN ECONOMIC, SOCIAL
RESEARCH ENTERPRISE, WHICH HAS
BECOME ENRICHED WITH BIOLOGICAL
ADDITIONS AS WELL.
BOTH TALK ABOUT BIOMARKERS AND
HOW THEY FIT.
BUT TO THIS SLIDE IN PARTICULAR
WE HAVE BEEN ABLE TO ESTABLISH
PARTNERSHIPS ACROSS THE WORLD,
AROUND HARMONIZED COGNITIVE
ASSESSMENT PROTOCOLS.
AND TAKING ADVANTAGE OF THE WAY
IN WHICH SAMPLING FROM DIVERSE
POPULATIONS IN THE
REPRESENTATIVE STUDY HRS IN THIS
STUDY, BUT THE FAMILY OF THESE
STUDIES ACROSS THE WORLD SHOWN
ON THIS MAP GIVES ACCESS WITH
HARMONIZED STANDARDS TO THE VAST
MAJORITY OF THE WORLD'S
POPULATION AND GIVING US NEW
INSIGHTS INTO THIS GLOBAL
PROBLEM OF ALZHEIMER'S RELATED
DMENSS.
THIS NEXT SLIDE ILLUSTRATES THE
INCREASING UNDERSTANDING OF THE
WAY IN WHICH THE EXPOSOME THE
ENTIRE UNIVERSE THAT IMPINGES ON
US AS WE AGE ARE IMPORTANT IN
IDENTIFYING RISK FACTORS FOR
ALZHEIMER'S DISEASE.
NOW LOOKING AT A MORE SYSTEMIC
WAY OF THE ENVIRONMENTAL INPUTS
INFLUENCING ALZHEIMER'S.
NEXT, PLEASE.
THIS NEXT SLIDE IS AN
ILLUSTRATION OF THE IMPACT
COLLABORATEORY.
IT WAS TO IMPROVE THE CARE AND
HEALTH OUTCOMES OF PERSON LIVING
WITH DEMENTIA.
IT IS THE LONG-TERM CARE DATA
COOPERATIVE PUT TOGETHER A
CONSORTIUM OF MORE THAN 2,000
NURSING HOMES ACROSS THE COUNTRY
IN DIVERSE AREAS REPRESENTING
OVER HALF A MILLION RESIDENTS
LINKING WITH MEDICARE CLAIMS
DATA AND PROVIDING ALZHEIMER'S
RESEARCH.
THIS PROVIDED ENORMOUSLY USEFUL
WAS IN THE COVID EPIDEMIC IT WAS
POSSIBLE TO USE THIS NETWORK TO
IDENTIFY EARLY THE VERY
VULNERABLE POPULATIONS IN
NURSING HOMES, WHAT HAPPENED TO
THEM IN EXPOSURE TO THE COVID
VIRUS ITSELF AND WHEN THE
VACCINE BECAME AVAILABLE VERY
QUICKLY DOCUMENT THE SAFETY OF
VACCINES IN NURSING HOMES.
A GOOD PORTION OF NURSING HOMES
WERE SADLY AFFLICTED WITH
COGNITIVE IMPAIRMENTS IT BECOMES
IMPORTANT FOR THE FOUNDATION.
NEXT SLIDE.
I WILL JUST BRIEFLY MENTION A
NEW PART OF THE ALZHEIMER'S
RESEARCH ENTERPRISE.
THE NIH CAMPUS.
TAKE ADVANTAGE OF THE INTRAMURAL
PROGRAMS ACROSS ALL OF THE NIH
INSTITUTES, IN FACT, PROVIDE
SOME UNIQUE RESOURCES WE WANT TO
TAKE FULL ADVANTAGE OF IN
ADDRESSING ALZHEIMER'S RELATED
DEMENTIAS AS WELL.
A PHYSICAL PRESENCE ON THE
CAMPUS, TAKING ADVANTAGE OF ALL
OF THE NIH INSTITUTES IN A
VARIETY OF WAYS WITH THE MANTRA
BEING TO SERVE AND PROVIDE
RESOURCES TO THE FULL COMMUNITY
AS IT PROVIDES CUTTING EDGE
RESEARCH AND CREATE A MAP OF
DIFFERENT BRAIN CELL TYPES WITH
ROBOTICS AS ESTABLISHED BY THE
LARGE LIBRARIES OF IPS STEM
CELLING WITH INDIVIDUALS
AFFECTED WITH DISEASE OR NOT
INTRODUCING DISEASE CAUSING INTO
THE STEM KELS AND THE CAUSAL
RELACESHIP THE DETERMINANTS.
JUST AN ILLUSTRATION OF WHAT WE
CAN DO WHEN THE WILL IS THERE.
THIS WAS DURING THE TIME OF
COVID WE WERE ABLE TO GET WITH
CONGRESSIONAL AND NIH SUPPORT
PERMISSION TO ESTABLISH A
TEMPORARY VERY MUCH IN QUOTES
RESOURCE CENTER ON CAMPUS WHERE
SPACE WAS QUITE LIMITING.
IN MAY THE TRUCKS PART THE PARTS
OF THE BUILDING SYNTHESIZED IN
FLORIDA, BY JULY THEY WERE
ASSEMBLED BY SEPTEMBER YOU CAN
SEE THE OPENING WITH ROYE ROY
BLUNT WITH HIS NAME IN THE
DEDICATION OF THE FACILITY.
THIS SHOWS THE ALREADY 10
DIFFERENT NIH INSTITUTES WHOSE
STAFF AND IDEAS HAVE BEEN
BROUGHT TO BEAR ON THE CARD
RESEARCH ENTERPRISE WITH THIS
LARGE ARRAY OF COLLABORATING
ORGANIZATIONS, ACADEMIC, PRIVATE
SECTOR, AND PHILANTHROPIC.
NOW TO TURN TO SOME OF THE
EXAMPLES OF RESEARCH PROGRESS.
ULTIMATELY OF COURSE ONE OF THE
IMPORTANT OBJECTIVES HAS BEEN TO
ESTABLISH DISEASE MODIFYING
TREATMENTS FOR ALZHEIMER'S.
2023 AND 2024 SAW THE FIRST
APPROVALS BY FDA OF
INTERVENTIONING TO TREED THE
UNDERLYING PROCESS OF DISEASE.
THESE WERE ANTI-AMYLOID
ANTIBODIES.
GOT FULL APPROVAL.
LANDMARK EVENTS THAT ARE
IMPORTANT FOR THE FIRST TIME
AFFECT TREATMENTS AS WELL AS THE
UNDERLYING BIOLOGY OF DISEASE.
THE EFFECTS ARE NOT NEARLY WHAT
WE NEED IN TERMS OF DEGREE OR
MAGNITUDE AND ACCOMPANIED BY
EXPENSE AND ADVERSE EFFECTS AS
WELL.
WE ARE AT A POINT OF BEING
EXCITED AND REINVIGORATED BY
THESE SUCCESSES BUT AT THE SAME
TIME REDEDICATED TO TARGET
MULTIPLE COMPLEX IN INDIVIDUALS
WITH MULTIPLE PATHOLOGIES
CONTRIBUTING TO DISEASE.
GENETICS, JUST TO SAY BACK 10 OR
15 YEARS AGO WE KNEW OF JUST 10
GENES THAT WERE RISK FACTORS FOR
ALZHEIMER'S DISEASE.
THERE ARE NOW MORE THAN 80.
THEY FALL INTO PATHWAYS,
INFLAMMATION, LIPD METABOLISM,
AND HAVE BECOME IMPORTANT WAYS
IN WHICH WE HAVE DIVERSIFIED THE
TARGETS FOR TRANSLATION INTO
CLINICAL STUDIES.
I WANT TO POINT OUT ONE OF THE
EXAMPLES THAT REFLECTS BOTH THE
IMPORTANCE OF DOING RESEARCH ON
DIVERSE POPULATIONS, OF GLOBAL
POPULATIONS AND STUDYING
GENETICS AT LARGE SCALE.
SO THERE ARE RARE SET OF
INDIVIDUALS WITH AUTOSOMONAL
DEMPLED BY GENETIC MUTATIONS BY
THE AMYLOID OR PRE -- GENES
WHICH PROCESS THE AMYLOID
PROTEIN.
INDIVIDUALS AFFECTED TRAGICALLY
HAVE ONSET IN THEIR 30s AND 40s.
EVERYONE HAS A 50% IN THE
FAMILIES INHERITING THE DISEASE
CAUSING GENE.
THE LARGEST SUCH FAMILY IN IS
COLOMBIA.
THIS IS A SERIES OF STUDIES
CARRIED OUT SUPPORTED BY NIH TO
LOOK FOR FURTHER INSIGHTS INTO
THE DISEASE AND ILLUSTRATE THE
IMPORTANT IDENTIFICATION OF RARE
PROTECTIVE FACTORS.
APOE IS ONE VARIANCE OF THE
COMMON RISK FACTOR FOR
ALZHEIMER'S.
A RARE VARIANT WAS DISCOVERED
WHEN IT WAS NOTED FIRST ONE
WOMAN, SHE INHERITED THE
DISEASE-CAUSING DISEASE OF
ALZHEIMER'S DISEASE IN THIS
FAMILY FOR OVER 20 YEARS BEYOND
EXPECTED DISEASE HAS NO DISEASE
AT ALL.
IT WAS LATER DETERMINED SHE HAD
EXTENSIVE AMYLOID IN HR BRAIN
BUT NOT TAO.
SHED A APOE.
THERE WAS ANOTHER GENETIC
MUTATION, A MAN IN THAT SAME
FAMILY WHO HAD A DIFFERENT
MUTATION IN THE GENE THAT ALSO
PROVIDE PROTECTION.
IN THIS CASE THROUGH ANOTHER
GENETIC MECHANISM, ALL FOCUSING
ON THE SAME PATHWAYS.
NOW IT IS ALREADY AT THE POINT
WHERE THE USE OF POTENTIALLY
DOMINANT PROTECTIVE GENES CAN BE
INTRODUCED INTO THE BRAIN IN THE
FORM OF VIRAL VECTORS.
WE HEARD ABOUT SOME OF THESE
INITIATIVES IN THE FIRST
CLINICAL TRIALS OF SUCH
ENTERPRISE ARE OCCURRING USING
THE APOE2 OR APOE CHRIST CHURCH
INTRODUCED THE BRAINS OF THE
INDIVIDUALS AT RISK FOR
ALZHEIMER'S DISEASE.
BIOMARKERS ARE CRITICAL.
UNTIL RECENTLY WE ALL DIAGNOSED
ALZHEIMER'S DISEASE ONLY AT
DEATH.
THE DIAGNOSE IN LIFE WAS
PROBABLE ALZHEIMER'S DISEASE.
IN FAST YEARS THIS HAS CHANGED
DRAMATICALLY, BEGINNING WITH
FIRST THE IDENTIFICATION OF
AMYLOID IN THE LIVING BRAIN
THROUGH PET SCANNING AND THEN
IDENTIFY TAO, AND BIOMARKERS AND
FLUID AND CSF AND MOST RECENTLY
IN BLOOD.
ONE GENERATED THROUGH NIH
FUNDING HAS GONE INTO THE PUBLIC
DOE NAME, PRECIVITYAD2.
NOW WE BIOMARKER SUCH AS
AMYLOIDS AND T PD43.
WE WILL LOOK AT INDIVIDUAL WITH
AT RISK TO USE BIOMARKERS TO
LOOK IN MORE DETAIL PRECISELY
WHAT IS GOING ON IN THE BRAIN.
IN ADDITION DATA TOOLS, DIGITAL
BIOMARKERS ARE PROVING TO BE
EXTRAORDINARILY EFFECTIVE AS
WELL IN IDENTIFYING, DIAGNOSING
INDIVIDUALS AT RISK OR WITH
EARLY OR PROGRESSIVE CHANGES OF
DEMENTIAS.
ONE RECENT REPORT, AN
ILLUSTRATION OF BASIC SCIENCE AT
AN EVER INCREASING PACE IS BEING
TRANSLATED THE BRAIN ATLAS
CONSTRUCTED THROUGH THE BRAIN
INITIATIVE AT NIH.
A PRODUCTIVE ENTERPRISE
IDENTIFIED AT THE CELLULAR
LEVEL, THE INDIVIDUAL BEHAVIOR
OF MORE AND MORE COMPLEX OF THE
UNDERSTANDING OF THE NETWORKS
WITHIN THE NORMAL BRAIN.
THE INVESTIGATORS INVOLVED THERE
WERE RAPIDLY ABLE TO TRANSLATE
THIS INFORMATION INTO THIS CASE
THE SEATTLE ALZHEIMER'S DISEASE
BRAIN CELL ATLAS.
LOOKING AT THE BRAINS OF
INDIVIDUALS IN VARIOUS STAGES OF
PREROGATIVE DISEASE.
LOOKING AT QUANTITYTIVE
NEUROPATHOLOGY TO IDENTIFY
SPECTRUM DISEASES AND IDENTIFY
THE SPECIFIC CHANGES IN NEURAL
CELL TYPES AND GLIAL POPULATIONS
THAT ACCOMPANY DISEASE
IDENTIFYING AN EARLY, SLOW AND
SILENT PHASE WHERE SYMPTOMS ARE
SMALL, AMYLOID PLAQUES ARE
INCREASING IN NUMBER, BUT
SMALLER IN SIZE.
ACCELERATED STAGE WHERE MANY
THINGS CHANGE WITH FORMATION TAU
AND TANGLES.
WE CAN IDENTIFY ERR AND LATE
STAGE DISEASE AND TARGET.
ONE OF THE EARLY CHANGES OCCURS
IN THE SLOW SILENT PHASE IS LOSS
OF INHIBITORY NURNS.
THE EXCITATORY NURNS.
IT MAY BE DISLIB RUM, LOSS OF
INHIBITORY NURNS BECOME A RISK
FACTOR.
THESE ARE EMERGES RAPIDLY FROM A
MORE BASIC UNDERSTANDING OF WHAT
HAPPENS AT A CELLULAR AND
NETWORK LEVEL WITHIN THE BRAIN.
NUEURONS A FEW YEARS AGO, THERE
WAS CONDUCTED AN AHRQ AND
NATIONAL ACADEMIES SURVEY OF
WHAT WE KNEW ABOUT PREVENTIVE
MEASURES THAT WOULD ALLOW
RECOMMENDATIONS FOR PUBLIC
HEALTH APPLICATION.
AT THAT TIME A REPORT JUST IN
2017 THE ANSWER WAS THERE WAS
NOT SUFFICIENT DATA, NO DATA OF
SUFFICIENT QUALITY TO WARRANT
RECOMMENDATION OF PREVENTION.
THIS HAS CHANGED, AGAIN CHANGED
RAPIDLY IN THESE PAST YEARS.
THE FIRST WHICH THE
DEMONSTRATION IN A STUDY CALLED
SPRINT.
AGGRESSIVE OR SBNSIVE CONTROL OF
BLOOD PRESSURE WAS CAPABLE OF --
MORE RECENTLY, HERE IS AN
KPAMPLT OF ANOTHER.
HEARING DEFICITS WERE A RISK
FACTOR FOR ALZHEIMER'S DISEASE.
HERE A CLINICAL TRIAL WAS
SUPPORTED TO CORRECT HEARING
DEFICIT IN INDIVIDUALS HIGH RISK
FOR ALZHEIMER'S DISEASE WITH A
DEMONSTRATION OF CORRECTING THAT
DEFECT, IN FACT, DECREASED THE
RISK OF COGNITIVE DECLINE AND
THESE STUDIES WILL CONTINUE
ALONG WITH OTHERS.
I WILL RELATE TO IN SUMMARY
SLIDES.
A LARGE NUMBER OF PREVENTIVE
STRATEGIES BE APPROACHED.
IN THIS SLIDE JUST TO NOTE THAT
THE EARLY PHASE STUDIES CARRIED
OUT TO IDENTIFY DRUG CANDIDATES
HAVE IDENTIFIED SOME 22
SUPPORTED BY NIH RESEARCH IN
CLINICAL DEVELOPMENT PHASE ONE
AND PHASE TWO TRIALS.
AT THE BOTTOM OF THE SLIDES ARE
NOTED SOME TARGETS.
AMYLOID BETA, APOE, LIPIDS, ALL
OF WHICH ARE IMPORTANT AS WE
LOOK TO PERSONAL IDES THE
PROCESSES THAT ARE GOING ON IN
EACH INDIVIDUAL AND ADDRESS THEM
WITH PREVENTIVE AND THERAPEUTIC
INTERVENTIONS.
ALL TOLD 460 TRIALS ARE ACTIVE
THAT NIA IS SUPPORTING.
THIS INCLUDES PREVENTION AND
TREATMENT AND DEMENTIA CARE AND
CAREGIVERING.
WE'LL LOOK IN DETAIL.
PHARMACOLOGIC INTERVENTIONS.
I WANT TO EMPHASIZE THE DATA IN
TARGET AND PROCESSES.
AMYLOID IS 6 OF 54 TRIALS.
NOW INFLAMMATION, METABOLISM,
BIOGENETICS, ALL ARE TARGETS OF
THESE INTERVENTIONS.
AND PHASE TWO THREE AND FOUR,
AMYLOID IS FOUR, THE OTHER NINE
ARE LOOKING AT VASCULATURE AND
WE ARE LOOKING AT EXERCISE,
NUTRITION, COGNITIVE TRAINING
AND COMBINATION, SOME
COMBINATIONS.
INTERVENTIONS INCREASING ON THE
HORIZON.
# FOR DEMENTIA CARE AND
CAREGIVERING, WE HAVE PEOPLE
LIVING WITH DEMENTIA, LEARNING
HOW TO INCREASE THEIR QUALITY OF
LIFE AND THOSE WHO CARE WITH
THEM AND FOR THEM IS IMPORTANT.
JUST ILLUSTRATING WHAT I HAVE
SAID IN A KRCRYPTIC WAYS.
OUR APPROACH HAS BEEN TO DATE TO
USE TREATMENT, TREATMENT A OR
TREATMENT B AND THAT WHOLE
POPULATION SINCE WE HAVE BEEN
ABLE TO UNDERSTAND SUBSET
POPULATIONS.
IN THE NEXT SLIDE YOU SEE WE
HOPE TO BE AT THIS JUNCTURE
UNDERSTANDING WHAT IS GOING ON
IN BRAINS OF INDIVIDUALS AND
TARGETING PREVENTIVE
INTERVENTIONS AND TREATMENTS TO
THEM.
BY WAY OF SUMMARY, DIAGNOSTICS
FROM 2016 TO 2024 THE INCREASE
OF TECHNOLOGIES AND RESOURCES
HAVE BECOME AVAILABLE.
WE MOVE FROM THE AMYLOID PET
AGENTS TO TAU PET AGENTS.
SYMPTOM MANAGEMENT.
THE EARLY CHOLINESTERASE
INHIBITORS AND NOW WE ADDED
DRUGS THAT ARE IMPORTANT IN
INDIVIDUALS WITH SYMPTOMS OF
ALZHEIMER'S, AGITATION AND
DEPRESSION BEING TARGETED AS
WELL.
DISEASE MODIFYING THERAPIES.
WE HAVE GONE FROM NONE TO TWO
FDA APPROVED BY TRADITIONAL
APPROVAL AND MANY ADDITIONAL NOW
IN THE PIPELINE LOOKING FORWARD
WITH GREAT OPTIMISM TO HAVING
NEW EFFECTIVE INTERVENTIONS.
RISK REDUCTION NONE, INTENSIVE
BLOOD PRESSURE CONTROL, HEARING
INTERVENTIONS.
MOST DETERMINANTS TAILOR
LIFESTYLE TO RISK FACTORS PROVE
TO BE EFFECTIVE.
MULTIVITAMIN USE HAS BEEN SHOWN
TO POTENTIALLY UTILITY IN
DECREASING A RISK OF
ALZHEIMER'S.
FOR CARE AND CAREGIVING, TWO
EARLY STAGE TWO HAVE TRANSLATED
TO IMPLEMENTATION IN LARGE SCALE
WITH POPULATIONS IN NEED.
ONE OF THE LIMITING FACTORS AND
CRITICAL COME PONTS OF RESEARCH
IN ALZHEIMER'S HAS BEEN HEROIC
PARTICIPATION OF HUMAN
PARTICIPANTS IN THESE STUDIES
AND CHALLENGE WITH RECRUITING
WITH DIVERSITY IN THESE STUDIES
HAS BEEN ADDRESSED WITH SOME OF
THE ONLINE RESOURCES THAT HAVE
BEEN MADE AVAILABLE AS WELL AS
NEW TRACKING SYSTEMS TO IMPROVE
OUR ABILITY TO RECRUIT AND TRACK
OUR RECRUITS AND OPTIMIZE EARLY
STAGE OF THESE STUDIES
RECRUITING PEOPLE INTO TRIALS.
THERE ARE MANY OPPORTUNITIES FOR
INPUT.
THE NEXT LARGE SUMMIT COMING UP
IN MARCH WILL BE ADDRESSING THE
ALZHEIMER'S RELATED DEMENTIA
SUMMITS AND ENCOURAGE ANY AND
ALL TO PARTICIPATE WITH HUNDREDS
OF LEADING SCIENTISTS FROM
AROUND THE WORLD AND THOUSANDS
ONLINE PROVIDING INPUT AND
UPDATES AND OPPORTUNITIES AND
GAPS THAT WILL DRIVE RESEARCH IN
THE YEARS TO COME.
AND THIS JUST A LIST OF THE VERY
MANY WAYS WE HOPE YOU CAN STAY
IN TOUCH AND PROVIDE INPUT INTO
WHAT WE ARE DOING.
THANKS.
>> RICHARD, THANK YOU SO MUCH.
AND THIS IS NOW OPEN FOR
QUESTIONS.
>> OKAY.
WHO WANTS TO GO FIRST?
IT IS ALWAYS HARD TO BE THE LAST
PERSON, RICHARD.
>> THARNGS FOR A VERY
COMPREHENSIVE PRESENTATION.
MY QUESTION IS RELATED TO REAL
WORLD DATA AND HOW IT CAN GUIDE
RECRUITMENT OF DIVERSE
POPULATIONS.
WHAT ARE THE CURRENT PLANS FOR
ITS USE?
>> THAT'S A GREAT QUESTION.
SO WE ALREADY HAVE, AS I HAVE
MENTIONED REAL WORLD IN DATA
POPULATIONS THE NURSING HOME
NETWORK AS ONE OF THE EXAMPLES.
THE DATA COUNTS ENTERPRISE WHICH
YOU'VE BEEN HEARING ABOUT FROM
MONICA.
IT IS THE FUTURE OF THIS KIND OF
RESEARCH IN WHICH RECRUITING
PEOPLE FROM DIVERSE PARTS OF THE
COUNTRY INTO POPULATIONS TO
ALLOW US TO IDENTIFY THOSE FROM
REAL WORLD DATA WHO ARE AT RISK
AND ARE PARTICIPANTS FOR
RECRUITMENT IN THE TRIALS
WORKING WITH EXISTS RESOURCES
SUCH AS ALL OF US.
WE HAD LINKAGES ESTABLISHED VERY
EFFECTIVE TOGETHER WITH CMS
LINKING MEDICARE DATA TO HEALTH
RECORDS AND USING THESE AS A
SAMPLING FRAME TO HELP US
RECRUIT, THIS TOPIC OF REAL
WORLD DATA AND HOW WE CAN MOST
EFFECTIVELY AND PAIR WITH
MONICA'S ABLE LEADERSHIP IN THE
FIELD IS IMPORTANT PART OF
ADDRESSING RECRUITMENT INTO
STUDIES AND TRACKING WHAT
HAPPENS ONCE THERE ARE OUTCOMES.
IT IS ONE THING TO CARRY OUT
CLINICAL TRIALS AND ANOTHER HOW
THEY FARE WHEN APPLIED IN BROAD
POPULATIONS.
>> GISELLE, YOU ARE NEXT.
>> RICHARD, DR. HODES, THANK YOU
SO MUCH FOR THE INCREDIBLE WORK
YOU HAVE DONE AROUND AGING AND
ALZHEIMER'S DISEASE.
I WANTED TO ASK YOU ARE WE AT
THE POINT WHERE THERE'S -- WHERE
WE'RE READY FOR AN ALZHEIMER'S
MOONSHOT?
DO WE HAVE THE PIECES TOGETHER
TO ALLOW US TO SEE THE KINDS OF
ADVANCES AND LEAPS?
ARE WE READY TO SET A GOAL OF
ELIMINATING OR REDUCING THE RISK
OF ALZHEIMER'S BY A SET DATE?
IF WE ARE, WHY HAVEN'T WE DONE
SO ALREADY AND IF NOT, WHAT ELSE
DO WE NEED TO DO TO GET TO THAT
POINT?
>> I CERTAINLY AGREE THAT IS THE
GOAL.
IN FACT, IT HAS BEEN.
IT IS AN EXPLICIT GOAL IN THE
ALZHEIMER'S PLAN.
FIRST IT WAS TO IDENTIFY
EFFECTIVE TREATMENT AND
PREVENTIVE INTERVENTIONS BY
2025.
AND THAT PLAN HAS NOW BEEN
LEGISLATIVELY RENEGOTIATED.
BUT AS I ILLUSTRATED, THE FIRST
STEPS TOWARD IDENTIFICATION OF A
TREATMENT THAT HAS SOME EFFECT
IN TREATING ALZHEIMER'S,
IDENTIFICATION OF RISK FACTORS
AND IDENTIFICATION OF HELP AND
PREVENTION ARE CLEARLY IMPORTANT
HARD STEPS BUT NOT SUFFICIENT.
SO OUR COMMITMENTS NOW ARE
CERTAINLY AT THE MOONSHOT LEVEL
OF TAKING THE CONSIDERABLE
RESOURCES WE HAVE AND MAXIMIZING
AT EACH STAGE AS I TRIED TO
ILLUSTRATE, A SYSTEMATIC WAY OF
GOING FROM IDENTIFYING RISK
FACTORS, MOVING INTO PRECLINICAL
AND TRANSLATIONAL STUDIES AND
RESOURCES INFRASTRUCTURE ARE
THERE WITH EVERYONE WITH BRIGHT
IDEAS TO ACCELERATE THOSE IDEAS
INTO TREATMENTS.
WHY WE ARE NOT THERE YET IS A
SOURCE OF FRUSTRATION FOR ALL OF
US.
IT IS A CLEARLY CHALLENGING
PROBLEM.
THERE IS GROUNDS FOR GREAT
EXCITEMENT BASED ON THE ENORMOUS
ACCELERATION AND THE MEANS TO
TRANSLATE RAPIDLY.
WE ARE IMPATIENT.
WE ARE DISSATISFIED BUT EXCITED
AND ENCOURAGED FOR WHAT IS TO
COME.
SURE APPRECIATE THE QUESTION.
>> RUSS, YOU ARE NEXT, PLEASE.
>> LET ME ECHO THE APPRECIATION
FOR THE AMAZING WORK THAT IS
BEING DONE AND I HOPE AMERICA
TAXPAYERS ARE WATCHING THE VIDEO
FEED BECAUSE THEY WILL FEEL THEY
ARE GETTING GREAT VALUE FOR
THEIR INVESTMENT IN THIS
RESEARCH.
MY QUESTION FOLLOWS ON
GISELLE'S.
AS WE LOOK AT HOW THIS CRISIS IS
GOING TO BE ADDRESSED, IT LOOKS
LIKE THE EASY FIXES OF TARGETING
TAU OR AMYLOID HAVE NOT PANNED
OUT AS ONE WOULD HAVE WISHED.
THIS SEEMS MORE LIKE A
PREVENTION DISEASE AT THIS POINT
THAN A REVERSAL DISEASE.
WITH ALL THE WORK BEING DONE ON
BIOMARKERS AND SURROGATE MARKERS
FOR DISEASE, WHAT ARE THE
PROSPECTS FOR VERY EARLY
DETECTION?
I'M BASED IN SEATTLE SO I'M
FAMILIAR WITH THE HEALTHY
POPULATIONS THAT DEVELOP
DEMENTIA TO DEVELOP THESE
MARKERS.
FROM THE AGING INSTITUTE'S
PERSPECTIVE, WHAT SORT OF THE
PROGRAMMATIC EMPHASIS HERE ON
EARLY DETECTION?
>> GREAT IMPORTANCE.
SO IT INVOLVES CON VERY JENS OF
NECESSARY COMPONENTS.
FIRST THE IDENTIFICATION AS YOU
ARE NOTING OF RISK FACTORS.
SOME OF THOSE RISK FACTORS WELL
KNOWN ALREADY, SUCH THINGS AS
CARDIOVASCULAR RISK, HEAD
TRAUMA.
OTHERS ARE FORMULATED
GENETICALLY, WHICH HELP IDENTIFY
THOSE AT HIGHEST RISK AS WELL.
AND THERE ARE MORE EXPLICIT OR
DIRECT BIOMARKERS JUST BEING
DEVELOPED ACTUALLY REFLECTING
PROCESSES GOING ON IN THE BRAIN
THAT ARE EARLY PRECLINICAL
STAGES OF THE PROCESSES.
THOSE ARE COMING ALONG AT A
DRAMATIC PACE TO THE POINT WHERE
IT IS CONCEIVABLE TO SCREEN VERY
LARGE NUMBERS OF INDIVIDUALS AT
RISK AND AT THOSE EARLY STAGES
IN CLINICAL TRIALS, PREVENTION,
PHA
PHARMACOLOGIC.
WE ARE AT THE POINT TO THE
DEGREE TO WHICH WE TOOK ALL THE
KNOWN STATISTICAL RISK FACTORS
WE COULD REDUCE ALZHEIMER'S.
IT THERE IS AN IMPORTANT
DISTWINS BETWEEN IDENTIFYING
SOMETHING AS A RISK FACTOR AND
SHOWING EM PEERICALLY MODIFYING
WILL HAVE THE ANTICIPATED
IMPACT.
THAT IS WHERE WE ARE NOW.
WE HAVE LARGE SCALE STUDIES
GOING ON IN INDIVIDUALS AT RISK
LOOKING AT PREVENTIVE MEASURES
THAT ARE IN SOME CASES
MULTIDETERMINANT.
LOOKING AT THE INDIVIDUAL AND
LOOKING AT EXERCISE PROGRAMS,
WEIGHT PROGRAMS AND NUTRITION.
WE ARE ADDING THE BIOMARKERS SO
WE DON'T HAVE TO WAIT FIVE, 10,
20 YEARS FOR ASYMPTOMATIC PEOPLE
TO SHOW COGNITIVE CHANGES BUT
ANTICIPATE BY STUDIES OF
BIOMARKERS.
THIS IS REALLY NEW STUFF.
WI HAVE BIOMARKERS THAT ARE
ACCESSIBLE ENOUGH, BLOOD,
INEXPENSIVE ENOUGH THAT BE
APPLIED TO LARGE POPULATIONS IN
CONCERT WITH INTERVENTIONS.
>> MY QUESTION IS A LITTLE
DIFFERENT.
ARE REREADY TO USE THE RISK
FACTORS TO IDENTIFY IN CLINICAL
TRIALS HIGHER THAN RISKED A
BASELINE TO IMPROVE THEIR POWER?
ARE WE READY TO TAKE A BLOOD
SAMPLE AND SAY YOU HAVE A 20%
CHANCE OF DEVELOPING ALZHEIMER'S
IN THE NEXT FOUR YEARS AND
THEREFORE YOU COME IN THIS TRIAL
BECAUSE YOU ARE IN A HIGH RISK
GROUP OR ARE WE NOT THERE YET?
>> NO, RUSSELL, ABSOLUTELY
THERE.
ALL OF OUR STUDIES NOW WHERE
INITIALLY INDIVIDUALS WHO MIGHT
BY COGNITIVE TESTING THOUGHT TO
BE AT AN EARLY STAGE OR FAMILY
HISTORIES WENT INTO STUDIES.
EARLY STUDY DAYS WE WERE
TREATING FOR ANTI-AMYLOID EVEN
IF WE DIDN'T KNOW THEY HAD IT.
THEN IN A PET SCAN, RIGHT NOW
OUR SCREENING PROCESSES FOR
INDIVIDUALS BLOOD BIOMARKERS AS
THE FIRST STEP.
THOSE INDIVIDUALS WHO SHOW
POSITIVE GO ON TO SCREENING FOR
ADMISSION INTO POLICY.
THAT IS ALREADY HERE.
>> GOOD.
THE ANALOGY IN MY FIELD MACULAR
DEGENERATION TRIALS WHICH
REQUIRED 5,000 PEOPLE SHOWED
VITAMIN SLOWED IT.
THE BANDWIDTH OF THAT STUDY IS
LIMITED.
I'M GLAD TO HEAR THAT BIOMARKER
WORK IS GOING TO GO IN CLINICAL
TRIAL DESIGN.
>> GREAT.
THANK YOU.
ELIZABETH, YOU ARE NEXT, PLEASE.
>> I HAVE A BASIC SCIENCE
QUESTION, BUT I DIDN'T KNOW
ABOUT THE CHRIST CHURCH VARIANT
FOR APOE.
I'M VERY CURIOUS WHETHER YOU
THINK -- I KNOW THIS IS A VERY
EXCITING AREA FOR ALZHEIMER'S
RESEARCH AND WHETHER YOU ARE
CONSIDERING REALLY FUNDING THAT
AREA TO DEVELOP AND SEE IF THIS
COULD SOMEHOW BE A THERAPY.
>> YEAH.
ABSOLUTELY.
SO AN AREA AROUND PROTECTIVE
VARIANTS AND THEIR BIOLOGY IS
VERY MUCH AT THE FOREFRONT.
AS I MENTIONED, THERE IS ONE
TRIAL, IN EARLY PHASE.
THEY MAY HAVE RESULTS AS EARLY
AS THE NEXT FEW MONTHS THAT IS
TAKEN EITHER -- SO APOE, THE
GENE WITH MULTIPLE ALLELES, THE
APOE4 IS THE HIGHEST ALLELE,
APOE2, IF YOU HAVE THAT VARIANT
SHOWS A DECREASED RISK.
THEN CHRIST CHURCH VARIANT.
TWO DIFFERENT STYLES OF A
GENETIC VARIANT ASSOCIATED WITH
LOWER RISK.
THERE ARE NOW TRIALS USED ADENO
VIRUS INTRODUCTION INTO THE
BRAIN OF CHRIST CHURCH APOE2 OR
COMBINATIONS OF THE TWO, FIRST
TO LOOK HOW WELL THEY PENETRATE
AND EXPRESSED AND IMPACT APOE4
AND AMYLOID.
WE ARE AT THE FOREFRONT.
EXTREMELY EXCITING TIME.
>> MARK, YOU ARE NEXT.
>> GREAT PRESENTATION.
I APPRECIATE THE NIA SUPPORTING
OTHER INSTITUTES IN A COMMON
GOAL TO FIGHT CEREBRAL VASCULAR
DISEASE.
IT IS A VASCULARTURE.
THE AMYLOID BOYT -- BETA
PARLIAMENTS ARE IN THE WE ARE
TALKING ABOUT BLOOD FLOW TO THE
BRAIN.
ONE OF THE DEFICITS IS IN
CEREBRAL FUSION.
RHETT -- RETINAL SCANS AND
DIMINISH BLOOD FLOW IN THE EYE,
NONINVASIVE TO THE CENTRAL
NERVOUS SYSTEM.
OR SOME WAY OF ADDRESSING BLOOD
FLOW BEFORE OVERT CLINICAL
PROBLEMS.
>> ABSOLUTELY.
IN THE BRAIN STUDYING BRAIN FLOW
HAS LONG BEEN IMPORTANT IN MRI.
WE HAVE BEEN LOOKING AT
MICROVASCULARTURE.
THE EYE HAS BECOME AN AREA OF
VERY IMPORTANT EMPHASIS FOR
MULTIPLE REASONS.
THERE HAVE BEEN FINDINGS OF
AMYLOID IN THE RETINA.
NOW WITH EVEN MORE SOPHISTICATED
COLLABORATION INCLUDING NEI FOR
MACHINE LEARNING APPLICATIONS TO
WHAT IS HAPPENING IN TISSUES
LIKE THE RETINA BEYOND SO FAR
THE INTUITIVE OBSERVATIONS OF
INDIVIDUALS.
VERY EXCITING.
NONINVASIVE, INEXPENSIVE, LARGE
POPULATIONS.
>> LET ME THROW OUT ONE OTHER
BIOMARKER.
WE WORK ON CADICIL, THE MUTATION
OF THE -- GENE.
IT IS IMPLICATED IN SMALL VESSEL
DISEASE.
THAT COULD BE ANOTHER BIOMARKER.
IT DOES GET IN THE BLOOD STREAM.
IT IS ONLY IN SMOOTH MUSCLES IN
THE BRAIN.
>> A NUMBER OF FACTORS IN THE
BLOOD AND THE BRAIN AND WHERE
THE BLOOD MAY BE REFLECTING WHAT
IS HAPPENING IN THE BRAN AND
IMPORTANT BIOMARKER AND THE
CHALLENGES UBIQUITOUS AND MAYBE
JUST THE SPECIES COMING FROM THE
BRAIN ARE IMPORTANT.
ANOTHER DIVERSION LOOKING INTO
EXTRA CELLULAR VESICLES, WHERE
ONE CAN TRACK THEIR ORIGIN, THE
ORIGIN OF THEIR VESICLES AND
LOOK AT THE MOLECULES IN THOSE
VESICLES IS THE NEAR SCIENCE
FICTION EXAMPLES HOW WE ARE
PURSUING TO UNDERSTAND BETTER
WHAT IS HAPPENING IN THE BRAIN.
>> THANK YOU.
GISELLE, DID YOU HAVE AN
ADDITIONAL QUESTION OR COMMENT?
>> NO.
I DIDN'T.
IS MY HAND STILL UP?
SORRY.
>> NO PROBLEM AT ALL.
>> I'M GOING TO TURN IT BACK TO
MONICA BECAUSE WE ARE JUST ABOUT
AT TIME.
>> THANK YOU SO MUCH.
OKAY.
I'M JUST GOING TO DO A QUICK
RECAP.
I THINK WE HAVE SOME SLIDES.
THANK YOU.
PT NEXT SLIDE, PLEASE.
OKAY.
WELL, WE ARE VERY PLEASED IN DAY
TWO OF OUR MEETING TO HAVE
HIGHLIGHTS SOME NIH PROGRAMS,
FIRST RESPONSIVE TO THE PEOPLE
WITH NEEDS WITH MAJOR PROGRESS
SINCE ITS INITIATION IN 2016 IN
RESPONSE TO THE CANCER MOONSHOT.
ALSO WITH THE ABCD STORY, YOU'VE
SEEN A CRITICAL INFRASTRUCTURE
PROJECT REACHING DATA MATURITY
NOW THAT ADDRESSES AN IMPORTANT
KNOWLEDGE GAP IN ADOLESCENT
BRAIN DEVELOPMENT AND HEALTH.
WHAT A CRITICAL TIME TO SEE A
RESOURCE LIKE THAT ARRIVE.
SO MANY THINGS THAT WE KNOW
ABOUT OUR CHANGING WORLD THAT
ARE BEING CAPTURED IN THE ABCD
STUDY.
AND ALSO A REVIEW OF PROJECT OF
THE NATIONAL PLAN TO ADRES
ALZHEIMER'S AND RELATED
DEMENTIAS.
TRULY COMPREHENSIVE DRIVE TO
CAPITALIZE ON ALL TECHNOLOGIES
AND PARTNERSHIPS TO UNDERSTAND
AND OVERCOME SUFFERING FROM
DEMENTIAS.
I APPRECIATED THE PRESENTATION
FROM DR. JULIE GERBERDING
OUTLINING OUR EVER GROWING
PARTNERSHIP WITH THE FOUNDATION
FOR THE NIH.
I PARTICIPATED IN THE BIOMARKER
CONSORTIUM QUITE A NUMBER OF
YEARS AGO WHEN IT FIRST ARRIVED.
IT IS AMAZING TO SEE HOW MUCH
FOUNDATION FOR NIH IS DOING FOR
US TODAY.
IT IS REALLY OUR PRINCIPAL
COLLABORATOR AS WE EXECUTE
PUBLIC-PRI
PUBLIC-PRIVATE-PERSON
PARTNERSHIPS.
I HOPE WITH THIS MEETING WE HAVE
ILLUSTRATED WITH VERY CLEAR
EXAMPLES BUT I'LL TELL YOU THERE
ARE MANY MORE, THAT NIH IS NOT A
COLLECTION OF 27 SILOS, BUT
INSTEAD A REALLY HIGHLY
COLLABORATIVE ENVIRONMENT THAT
IS GROUNDED IN FUNDAMENTAL
SCIENCE THAT FOCUSED ON
DELIVERING HEALTH AND ALSO ON
NURTURING THE NEXT GENERATION.
WITH THAT, THIS BRINGS US TO THE
CLOSE OF OUR DIRECTOR'S ADVISORY
COMMITTEE MEETING TODAY.
I JUST WANT TO THANK ALL OF THE
PEOPLE IN THE OFFICE OF THE
DIRECTOR HERE AT NIH, THE
SUPPORT TEAM THAT HELPED
COORDINATE THIS MEETING.
TO ALL OF THE SPEAKERS WHO I
THINK JUST DID SUCH A WONDERFUL
JOB AND MOST OF ALL TO THE
COMMITTEE MEMBERS.
WE'RE SO GRATEFUL FOR YOUR
GENEROUS AND KNOWLEDGEABLE
PARTICIPATION.
FINALLY, BEFORE I GAVEL US OUT,
I JUST WANT TO AGAIN EXPRESS MY
GRATITUDE TO EVERYONE FOR THE
INCREDIBLE PRIVILEGE OF SERVING
AS NIH DIRECTOR.
SO WITH THAT, I'M GOING TO BANG
THE GAVEL.
I WISH YOU ALL A WONDERFUL
HOLIDAY SEASON AND A VERY HAPPY
AND HEALTHY 2025.
SO MEETING ADJOURNED.