>> GOOD MORNING WELCOME TO THE
37th MEET FOR THE NATIONAL
CENTER FOR ADVANCING
TRANSLATIONAL SCIENCES ADVISORY
COUNCIL IS NOW IN SESSION.
I'LL GAVEL US IN.
>> LET'S BEGIN WITH ROLL CALL
PAUL HARRIS.
AMY KARLY.
KELLY McBERRY.
ROBIN MIDDLESTEIN.
KEITH MILLER.
>> HERE.
>> PAULA SHIREMAN.
>> WE HAVE ALL MEMBERS IN
ATTENDANCE AND NO AD HOCS
PARTICIPATING.
SO, OPEN SESSION IS ONE DAY,
TODAY.
THE ACTIVITY WILL BE THE
DIRECTOR'S REPORT AND WE'LL THEN
HAVE A 45 MINUTE LUNCH BREAK.
AFTER LUNCH WE HAVE A SPECIAL
TOPIC PRESENTATION AND
DISCUSSIONS AND THEN MOVE INTO
TWO UPDATES AND THE FIRST WILL
BE THE DIVISION OF CLINICAL
INNOVATION PRESENTED BY MIKE AND
THEN THE DRUG DEVELOPMENT
PARTNERSHIP PROGRAMS BY
CHRISTINE AND THEN CONCEPT
PRESENTATIONS THE FIRST BY THE
OFFICE OF DRUG DEVELOPMENT
PARTNERSHIP PROGRAMS AND THE
SECOND WILL BE PRESENTED BY THE
OFFICE OF SPECIAL INITIATIVES
AND THE LAST FOR THE DAY WILL BE
PRESENTED BY THE DIVISION OF
RARE DISEASES RESEARCH
INNOVATION.
ANNOUNCEMENTS TODAY, THIS IS A
HYBRID MEETING.
THOSE ON SITE ARE NOT LOGGING
INTO THE ZOOM NECESSARY.
AND IF YOU DO MAKE SURE YOU ARE
NOT CONNECTED TO YOUR AUDIO.
THE CONFERENCE ROOM IS CONNECTED
TO ZOOM AND THAT'S ATTENDEES
PARTICIPATING AND BEING SEEN.
IF YOU ARE PARTICIPATING
VIRTUALLY, PLEASE RAISE YOUR
HAND OR CHAT AND WAIT UNTIL
YOU'RE CALLED UPON TO PROVIDE
INPUT.
ALL PARTICIPANTS PLEASE ALLOW
SPEAKERS TO MOVE THROUGH THEIR
PRESENTATIONS.
NOTING YOUR COMMENTS AND/OR
QUESTIONS AND PRESENTING YOUR
COMMENTS WHEN CALLED UPON.
ANY VIRTUAL PARTICIPANTS MUST
ENSURE DURING THE MEETINGS --
EXCUSE ME, SO WE'RE USING
VIDEOCAST FEATURE.
THE AUDIENCE MEMBERS MAY SUBMIT
COMMENTS UNTIL OCTOBER 3 BY
USING THE E-MAIL FUNCTION
AVAILABLE ON THE NIH VIDEOCAST
SITE.
YOU HAVE AN E-MAIL ADDRESS NCATS
COUNCIL INPUT AT MAIL DOT NIH
DOTGOV.
IF YOU ARE NOT ON CAMERA WHAT
HAPPENS IS THE PREVIOUS SPEAKER
IS SPOTLIGHTED IN THE VIDEOCAST.
THE FUTURE MEETING DATES 2025
ARE JANUARY 30-31.
MAY 22 AND MAY 23, SEPTEMBER 18
AND 19 AND 2026, JANUARY 29 AND
MAY 21 AND 22 AND SEPTEMBER 17
AND 18 AND THE JANUARY MEETINGS
ARE VIRTUAL MEETINGS.
NOW I'M GOING TO HAND IT BACK TO
OUR I.C. DIRECTOR DR. JONI
RUTTER FOR OUR REPORT TO
COUNCIL.
>> A QUICK MIKE CHECK.
CAN YOU HEAR ME OKAY?
THANK YOU.
THIS IS THE FIRST TIME IN THE
SHADY GROVE CAMPUS AND I'M
LOVING THIS AND THE FOURTH
MEETING IS ALWAYS THE MEETING
THAT HAS SO MUCH ACTIVITY
RUNNING UP TO IT THOUGH IT
COVERS THE SUMMER PERIOD.
THERE'S A LOT OF ACTIVITY
HAPPENING IN WHAT WE DO BUSINESS
WISE FOR THE GRANTS MANAGEMENT
REVIEW.
I WANT TO GIVE A BIG ROUND OF
APPLAUSE AND SHOUT OUT TO OUR
COUNCIL TEAM FOR ORGANIZING AT
THE SAME TIME THEY'RE WORKING ON
THE REVIEWS AND GRANTS
MANAGEMENT WORK WE DO IN THE
DIVISION OF EXTRAMURAL AFFAIRS.
SO THANK YOU ALL.
THANK YOU FOR THAT IMPORTANT
WORK AND THERE'S QUITE A LOT TO
DISCUSS AND I'M NOT SEEING MY
SLIDES ON MY SCREEN SO I MIGHT
BE A LITTLE BIT DISCOMBOBULATED
BUT HOPEFULLY I CAN FIGURE OUT
HOW TO ADVANCE THEM.
OKAY.
I USUALLY DO A RECAP WHAT WE DO
FROM OUR LAST COUNCIL.
I'M NOT GOING THROUGH THE ENTIRE
SLIDE BECAUSE WE TALKED ABOUT
QUITE A LOT.
WELL GO OVER SOME OF THIS AGAIN
JUST AS RELATED TO UPDATES I
WANTED TO MENTION.
IN TERMS OF OUR BUCKET I'LL TALK
ABOUT THAT BUT WE HAVE 11 DAYS
LEFT.
WE STILL DON'T HAVE A BUDGET YET
BUT HOPING WE'LL HAVE A
CONTINUING RESOLUTION TO GET US
THROUGH BUT I'LL GIVE A SENSE OF
WHERE WE ARE AND TO REMIND YOU
THE LAST ROUND WE TOLD YOU FOR
THE BUDGET YEAR OF FY24 WE HAD A
$5 MILLION INCREASE IN OUR
ACTIVITIES THAT IS SUPPORTING
AND BOLSTERING OUR TEAM THERAPY
EFFORTS AND IN PARTICULAR
DISEASE CALLED MMA101 AND WE'RE
PUSHING FORWARD AND HOPEFULLY
WE'LL BE ABLE TO UPDATE YOU.
IT WON'T BE TODAY BUT WANTED TO
REMIND YOU.
AND WE TALKED ABOUT OUR
STRATEGIC PLAN.
I'LL GIVE AN UPDATE AS WELL
BECAUSE I'M EXCITED WE HAVE
PUBLISHED OUR STRATEGIC PLAN.
HANG TIGHT.
WE'LL TALK ABOUT THAT IN A
MINUTE.
ALL RIGHT.
TODAY WE ARE -- WE HAVE A
VARIETY OF OTHER PRESENTATIONS
AFTER ME BEFORE WE DO CONCEPT
CLEARANCES BUT I'M EXCITED TO
WELCOME -- YOU'RE WONDERING WHO
WE'RE GOING TO WELCOME TODAY.
OKAY.
HE HAD TO WALK OVER TO MAKE IT
WORK.
SO WE HAVE A SPECIAL
PRESENTATION TODAY FROM JANINE
PRITCHETT CLAY AND I'VE BEEN
WAITING FOR THE PRESENTATION AND
I'M EXCITED YOU'LL BE ABLE TO
HEAR IT TODAY.
I'M NOT GOING TO SAY MORE
BECAUSE I DON'T WANT TO STEAL
THEIR THUNDER BUT HOPE YOU'LL BE
AS IMPRESSED AS I AM IN THE WORK
THEY'VE BEEN DOING.
WE HAVE OTHER UPDATES IN TERMS
OF OUR PROGRAMATIC ACTIVITIES
WITH THE DIVISION OF CLINICAL
INNOVATION AND THE OFFICE OF
DRUG DEVELOPMENT PARTNERSHIP
PROGRAMS.
MIKE WILL BE PRESENTING FROM DCI
AND CHRISTINE AND KARLIE WILL BE
TALKING ABOUT THE OFFICE OF DRUG
DEVELOPMENT PARTNERSHIPS
PROGRAM.
WE HAVE SOME REALLY GREAT
PRESENTATIONS FOR YOU HERE TODAY
TOO.
I ALSO WANTED TO SHOW THIS GREEN
BOX UP HERE IS THE ONLY
REMAINING BOX WE HAVE NOT QUITE
FILLED IN TERMS OF OUR HIRES BUT
WE CONTINUE TO HAVE THE
EXPERTISE OF BECCA GIGER AS OUR
EXECUTIVE OFFICER.
WE'RE HOPING TO FINALIZE THE
ROLE OTHERWISE WE'RE SET IN OUR
LEADERSHIP AND I COULDN'T BE
MORE HAPPY.
WE'LL CONTINUE TO KEEP HUMMING
ALONG BUT AT A BETTER CLIP.
I'M SUPER EXCITED ABOUT THAT.
SO I ALSO WANT TO GO OVER
UPDATES AND I CIRCLED HER IN
GREEN BECAUSE THIS IS THE FIRST
COUNCIL THAT WE HAVE ANIKA
WAYMAN AS THE OFFICIAL DEPUTY
DIRECTOR OF NCATS.
I'D LIKE TO TURN IT OVER TO HER
TO INTRODUCE HERSELF AS THE
DEPUTY DIRECTOR AND SAY A FEW
WORDS BECAUSE SHE HAS A UNIQUE
ROLE HERE.
>> GOOD MORNING, EVERYONE.
THANKS FOR THE INTRODUCTION.
REALLY EXCITED TO BE HERE.
IT FEELS KIND OF STRANGE BEING
ON THE OTHER SIDE BUT I DID
ENJOY MY ROLE AS COUNCIL MEMBER
SO MUCH SO I WANTED TO DIG IN
FURTHER.
SO JUST A REMINDER OF MY
BACKGROUND AND WHY THE ROLE
SEEMS SO PERFECT IS I'VE HAD A
BLEND OF EXPERIENCES UP TERMS OF
MY BACKGROUND.
THAT ALL LENT ITSELF TOWARDS
TRANSLATIONAL SCIENCE.
I FEEL LIKE I'M ABLE TO LEVERAGE
ALL THAT AND I'M AN ENGINEER.
I DID RESEARCH IN BIO PHYSICS
AND WORKED IN THE PRIVATE SECTOR
AND IN GOVERNMENT.
STARTED A TRANSLATIONAL SCIENCE
UNDERGRADUATE PROGRAM AT UMBC
RIGHT WHERE I CAME FROM BEFORE
HERE AND EXCITED TO WORK
ALONGSIDE JONI.
IT'S BEEN REALLY INTERESTING
THUS FAR.
AS A COUNCIL MEMBER YOU SAW THE
GREAT WORK AND NOW I HAVE AN
OPPORTUNITY TO SEE IT AS A
DEEPER LEVEL AND IT'S
FASCINATING WORK.
I LOVE ALONG WITH THE STRATEGIC
PLAN WHERE NCATS IS GOING SO
IT'S EXCITING TO BE PART OF
NCATS AND THE PEOPLE ARE PAS
PASSIONATE AND COUNCIL REAL AND
AUTHENTIC AND I'VE ENJOYED MY
TIME SO FAR AND HAVE IDEAS AND
THOUGHTS HOW TO LEVERAGE COUNCIL
MORE.
I DON'T KNOW IF YOU GUYS FELT
THAT.
I FELT AT COUNCIL I WANTED TO
FIGURE OUT HOW TO DO MORE WHICH
IS WHY I THEN CAME TO NCATS BUT
I THINK AS A COUNCIL MEMBER
THERE'S ALSO OTHER OPPORTUNITIES
AS TO HOW COUNCIL COULD BE
LEVERAGED.
SO LOOKING FORWARD TO HELPING
NCATS IN TERMS OF THAT PIECE AS
WELL.
THANK YOU FOR THE WELCOME.
>> THANK YOU, ANIKA.
WELCOME.
SO THAT'S GOOD NEWS BUT NOW WE
HAVE TO SAY GOOD-BYE TO A
COUNCIL MEMBER ANNIE KENNEDY WHO
HAS BEEN A DYNAMIC PARTNER I
THINK WITH THE COUNCIL TEAM AND
MEMBERS AND HAVE BENEFITTED FROM
HER LEADERSHIP NOT JUST WITHIN
COUNCIL BUT EVEN IN THE BROADER
COMMUNITIES.
EVEN THOUGH SHE'LL BE ROTATING
OFF OUR COUNCIL, I KNOW SHE
WON'T BE FAR.
I ALSO WANT TO GIVE A ROUND OF
APPLAUSE FOR ANNIE AND HER WORK
ON COUNCIL.
THANK YOU SO MUCH.
I'LL GIVE YOU A FEW MOMENTS AT
THE END OF THE MEETING IF THAT'S
OKAY BUT IF YOU'D LIKE TO SAY
ANYTHING NOW, HAPPY TO HEAR
THOUGHTS OR WOULD LIKE TO
WAIT -- OKAY.
FAIR ENOUGH.
SO THEN ON TO THE BUDGET.
THIS IS WHERE THINGS STAND NOW
WITH OUR BUDGET.
ON I THINK IT WAS MARCH 11 THE
PRESIDENT'S BUDGET WAS RELEASED
AND REQUESTED $926.1 MILLION FOR
NCATS FOR FY25 A 0.3% INCREASE
OVER FY23.
AS YOU MAY RECALL IT'S A LITTLE
BIT FUNNY HOW IT WORKED OUT.
THE TIMING WAS TIGHT IN TERMS OF
THE WHEN THE PRESIDENT'S BUDGET
WAS RELEASED AND THE FY24
BUDGETS.
THE FY24 BUDGET WAS BASED ON
FY23 BECAUSE IT WAS JUST PRIOR
TO THE PASSAGE OF THE BUDGET.
THAT'S WHY IT MAY LOOK ODD AND
THE HOUSE AND SENATE PASSED
BILLS BUT NOT BY THE FULL
COMMITTEE.
THAT WILL HAVE TO HAPPEN BEFORE
OCTOBER 1 OR THERE WILL NEED TO
BE A CONTINUING RESOLUTION OR WE
MAY RUN OUT OF MONEY IN 11 DAYS
BUT HOPEFULLY THAT WON'T HAPPEN
AND WE'LL GET A C.R. TO PREVENT
A LAPSE IN FUNDING IN THE
GOVERNMENT AND KEEP US OPEN A
BIT LONGER.
THE HOUSE PROPOSED A CONTINUING
RESOLUTION THROUGH THE 28th THAT
HASN'T HAPPENED SO STAY TUNED
AND WE'LL KEEP YOU POSTED.
IF THERE'S A C.R. WE'LL TELL YOU
WHAT'S GOING ON WITH THE BUDGET
NEXT TIME WE MEET.
IF YOU'RE INTERESTED IN OTHER
ACTIVITIES ED TO -- RELATED TO
THE BUDGET SEE OUR QR CODE SHOWN
HERE AND THAT'S WHERE WE ARE IN
THE BUDGET.
ON TO OTHER NCATS RELATED
ACTIVITIES.
THERE WILL BE I THINK
HIGH-PROFILE AND INTERESTING
MEETINGS I WANTED TO
HIGHLIGHTED.
FIRST AN ASSAY GUIDANCE NETWORK
FOR HIGH THROUGHPUT SCREENING
AND IT'S BEEN A HUGE SUCCESS
FROM DAY ONE .
I THINK THEY KEEP GETTING BETTER
AND BETTER.
SO IF YOU HAVE A CHANCE TO
REGISTER FOR THESE PLEASE DO SO.
QR CODE IS HERE.
AND THESE ALREADY HAVE QUITE A
BIG REGISTRATION FOR THEM.
THEY'RE ALL VIRTUAL.
I HOPE YOU'LL BE ABLE TO ATTEND.
THE SECOND WORKSHOP WE'RE DOING
IN OCTOBER IS ON IN SILICO DRUG
DISCOVERY.
NAMS OR COMPLEMENTARY PREDICTIVE
MODELS ARE A BIG EMPHASIS AND
THE WORKSHOP WILL BE RELATED TO
IN SILICO DRUG DISCOVERY AND
THINK IT WILL BE COMPANION TO
THE NAM PREDICTIVE MODELS WE
DEVELOPED AS WELL.
THESE IDEAS GO HAND IN HAND BUT
THIS ONE SPECIFICALLY IS
FOCUSSED ON IN SILICO DRUG
DISCOVERY.
MUCH OF THE EFFORT WILL BE
AROUND A.I. AND MACHINE LEARNING
ETCETERA.
PLEASE, IF YOU'RE ABLE TO JOIN
IN CONSIDER REGISTERING THERE
TOO.
A FEW OTHER ACTIVITIES WE'VE
DONE OVER THE SUMMER -- I FEEL
LIKE THIS IS A REPORT OF WHAT I
DID OVER THE SUMMER LIKE BACK TO
SCHOOL.
BUT WE HAVE EVERY YEAR A GROUP
FROM JAPAN CALLED THE MANSFIELD
SCHOLARS.
IT'S A RESEARCH SCHOLARS
PROGRAM.
ABOUT A DOZEN DIFFERENT EARLY
CAREER SCIENTISTS FROM JAPAN GET
TOGETHER AND WANT TO COME TO THE
U.S. AND LEARN ABOUT
TRANSLATIONAL SCIENCE.
THEY ALWAYS COME TO NCATS.
IT'S ONE OF THEIR FAVORITE STOPS
SO THEY SAY.
IT'S A REALLY WONDERFUL EXCHANGE
PROGRAM WHERE THEY GET TO SEE
THE LABS AND HEAR FROM SOME OF
OUR TRANSLATIONAL SCIENTISTS ON
A VARIETY OF PROJECTS.
OUR GOAL IS TO SHARE BEST
PRACTICES.
I'M A BIG BELIEVER WE CAN DO
MORE WITH SCIENTIFIC DIPLOMACY
AND THE MORE RELATIONSHIPS TO
THE MORE ADVANCES WE CAN MAKE
AND THIS IS WHERE I LIKE TO
SUPPORT AND THEY'RE EAGER TO
COME TALK TO US.
THAT WAS ONE OF THE THINGS.
ANOTHER INTERNATIONAL GROUP THAT
CAME TO VISIT JUST YESTERDAY WAS
THE PARIS BRAIN INSTITUTE.
ORGANIZED BY THE FRENCH EMBASSY
THE COUNSELOR OF SCIENCE AND
TECHNOLOGY AND WE DISCUSSED
POTENTIAL COLLABORATIONS
OVERLAPPING WITH TRANSLATIONAL
SCIENCE AND ALSO A BIG INTEREST
IN THE BRAIN, HENCE THE TITLE,
BUT A LOT OF AREAS RELATED TO
NEUROLOGY AND DISORDERS.
AGAIN WE WANTED TO MEET WITH
THEM TO TALK ABOUT DIFFERENT
IDEAS AND WHERE WE MIGHT BE ABLE
TO PARTNER.
SOMETIMES THESE LEAD TO GOOD
PARTNERSHIP OPPORTUNITIES.
SOMETIMES THEY DON'T.
AS YOU ALL KNOW WITH ANYTHING WE
DO WITH COMMUNITY ENGAGEMENT
IT'S NOT NECESSARILY WE LOOK FOR
ONE TIME TO HAVE AN IMPACT BUT
IT'S THE COLLECTION OF ENGAGING
WITH INDIVIDUALS OVER TIME AND
BUILDING THE RELATIONSHIPS.
THAT'S WHAT MAKES IT SO
POWERFUL.
WE HOPE WE CAN GET RELATIONSHIPS
OUT OF THIS BUT NONETHELESS I
THINK WE MADE GOOD FRIENDS AND
WILL CONTINUE TO HAVE
INTERACTIONS WITH THESE GROUPS.
IN ADDITION WE DID AN A.I.
DEMONSTRATION FOR THE SENATE
LAST TIME WAS FOR THE HOUSE.
THIS TIME WE DID ONE FOR THE
SENATE IN JULY.
I HAD AN OPPORTUNITY TO DISCUSS
A LOT OF TO THE WORK WE'RE DOING
THROUGH 3-D MODELLING AND SOME
OTHER A.I. AND ML PROJECTS AND
GOT INTEREST FROM STAFFERS FROM
THE SENATE.
THESE ARE JUST PHOTOS FROM SOME
CONVERSATIONS WE HAD.
NCATS IS ONE OF TWO I.C.s THERE
THAT DAY NABIB OUR NEIGHBOR NEXT
DOOR AND A WONDERFUL
COLLABORATIVE OPPORTUNITY TO
TALK WITH THE SENATE AND IT'S
ALWAYS IMPORTANT TO TALK ABOUT
THE ACTIVITIES BECAUSE IN THE
SPACE OF A.I. BIO MEDICINE ISN'T
ALWAYS AT THE FOREFRONT.
IT MIGHT BE PHYSICS ENGINEERING
AND THE OTHER KINDS OF SCIENCES
THAT ARE DONE.
BUT FOR US HAVING A.I. AND M.L.
AT THE FOREFRONT OF TRANSPARENCY
AND DATA CAPTURE AND THESE
THINGS IS INCREDIBLY IMPORTANT
FOR US TO COMMUNICATE TO OUR
SENATE AND CONGRESS
CONGRESSIONAL MEMBERS ABOUT THE
IMPORTANCE FOR BIO MEDICINE TO
MAKE SURE WE'RE AT THE TABLE TO
MOVE THESE TOPICS FORWARD
TOGETHER.
SO THAT WAS A REALLY EXCITING
DAY AND MORE RECENTLY ON
SEPTEMBER 11 I HAD A WONDERFUL
OPPORTUNITY TO SPEND SOME TIME
WITH CONGRESSWOMAN DEGETTE WITH
THE CURES IDEA.
IT WAS PART OF WHAT NCATS IS
BUILT FROM AND IT TURNS OUT AS I
WAS SITTING IN HER OFFICE I KEPT
SEEING THIS BLANKET ON THE END
OF HER CHAIR.
TURNS OUT IT'S NOT A BLANKET,
IT'S A CAPE.
IT ALSO TURNS OUT A CHILD WITH A
RARE DISEASE MADE THIS CAPE FOR
THE CAN CONGRESSWOMAN AND IT
TURNS OUT MAX IS THE SON OF LISA
SHIELD ON OUR BOARD.
IT WAS NICE TO SEE THAT COME
FULL CIRCLE.
HE MADE THE CAPE FOR THE
REPRESENTATIVE AND IT WAS A
WONDERFUL COMING TOGETHER OF
THESE THINGS.
IT WAS A GREAT CONVERSATION AND
AGAIN THIS IS ONE OF THOSE
THINGS WHERE WE HOPE WE CAN HAVE
CONTINUING CONVERSATIONS AND
BUILDING RELATIONSHIPS AND BE
ABLE TO TELL MORE STORIES OF
WHAT WE'RE DOING AT NCATS.
AND THEN ANOTHER IMPORTANT
MEETING WE HAD OVER THE SUMMER
WAS GETTING TOGETHER WITH THE
FDA AS WELL AS CMS LEADERSHIP
AND NIH THIS IS THE GROUP OF
PEOPLE AT THE MEETING AT THE FDA
AND IT WAS FOR THE PURPOSE OF
HOW THE AGENCIES CAN THINK ABOUT
BETTER COORDINATION IN THE SPACE
OF RARE DISEASES.
AND SO DURING THE CONVERSATION
WE HAD A VARIETY OF TOPICS WE
DISCUSSED AND AT THE BEGINNING
WE HAD A LIST OF TOPICS ON THE
BLACK AND IN RED AT THE END OF
THE CONVERSATION IS WHERE WE
STARTED TO CHANGE THINGS.
THIS IS WHERE WE WANT TO SKATE
TO THE PUCK AND CONTINUE TO MOVE
FORWARD.
IT WAS AN EXCITING MEETING.
SOME THINGS WE ARE DOING TO MOVE
FORWARD ARE ON THE LIST AND OVER
THE NEXT COUPLE OF YEARS YOU'LL
START TO SEE MORE OF THAT
FRUITION COME TO HAPPEN.
IN ADDITION TO OUR PARTNERSHIP
WITH THE FDA AND CMS IS THE
TRANSLATIONAL SCIENCE
INTERAGENCY FELLOWSHIP PROGRAMS.
THEY SPEND 18 MONTHS AT NCATS
AND FDA.
IT'S INTERMIXED.
GENERALLY SPEAKING THAT'S THE
POINT OF THE WHOLE PROGRAM AND
THE IDEA IS DO UPSCALE PEOPLE
WHO ARE IN REGULATORY SCIENCE
WITH TRANSLATIONAL SCIENCE OR UP
SKILL PEOPLE TO SCIENCE AND
BRING THEM INTO A FELLOWSHIP
PROGRAM AS THE PEOPLE GO OUT IN
THE WORLD AND GET THEIR NEXT FEW
JOBS, THEY'LL BE ABLE TO BRING
THAT EXPERIENCE WITH THEM.
THIS IS IS ONE OF THE PREMIER
PROGRAMS WE HAVE.
IT'S VERY SMALL BUT I'M REALLY
EXCITED ABOUT THIS PROGRAM.
THESE ARE THREE INDIVIDUALS WHO
JUST GRADUATED FROM THE PROGRAM
VERY RECENTLY.
AND ONE IS GOING INTO A BIO TECH
COMPANY.
ONE IS STAYING AT NCATS AND ONE
GOING TO THE FDA.
I'M EXCITE ABOUT THE PROGRAM AND
WE HAVE A FEW MORE COMING I HOPE
TO TALK ABOUT IN THE FUTURE AND
ONE TOPIC IS IN THE SPACE OF
NEWBORN SCREENING.
THIS IS A BIG AREA FOR NCATS
WE'RE INTERESTED IN IN MOVING
THE NEEDLE.
THERE'S BEEN TOPICS THROUGH THE
CTSA SCREENING IN NEWBORN
SCREENING SETTING THE STAGE FOR
WHY IT'S SO IMPORTANT.
THROUGH THE COMMON FUND PROGRAM
AND LAUNCHED A NEW COMMON FUND
PROGRAM THAT IS LIKE A VENTURE
COMMON FUND PROGRAM.
THEY'RE MEANT TO BE SMALLER BUT
MIGHTIER AND IT'S MEANT TO BE A
LITTLE BIT OF SEED MONEY CAN
HELP MOVE IT AND ONE TOPIC THAT
GOT SELECTED IS AROUND NEWBORN
SCREENING AND THIS TOPIC WAS
THOUGHT OF AND PRESENTED TO THEM
FROM OUR DEAR DRI DIRECTOR AND
THEY'LL BE LEADING THE PROGRAM
FOR NIH BUT PARTNERS WILL BE
NICHD AND DIANA BIANCHI WILL BE
PART OF THIS AND AN INVESTMENT
FOR FIVE YEARS SHOWING SCREENING
AS A POTENTIAL WAY OF A NEW
PARADIGM OF DOING NEWBORN
SCREENING.
WE'RE DEMONSTRATING FEASIBILITY
FOR A SMALLER PROGRAM BUT MAYBE
HOW THAT COULD BE SCALED TO AN
ACTION PROGRAM.
AND IMPORTANTLY EQUITABLE ACCESS
AND THEN HOW WE CAN THEN DRIVE
THE PACE OF THERAPEUTIC
DEVELOPMENT BASED ON THE
DIAGNOSTIC DEVELOPMENT WE HAVE.
AS YOU KNOW MANY PEOPLE WITH
RARE DISEASES HAVE A LONG
DIAGNOSTIC ODYSSEY IF WE CAN
SHORTEN THE DIAGNOSTIC ODYSSEY
IT MEANS WE'LL HAVE A LONGER
TREATMENT ODYSSEY LIKELY AND
NEED TO BE ADD THE READY TO
THINK HOW TO MOVE TREATMENT FOR
RARE DISEASES.
I THINK THIS IS A VERY EXCITING
PROGRAM TO PUSH THE ENVELOPE IN
THE SPACE.
STAY TUNED ON THAT.
ANOTHER EXCITING PROGRAM WE HAVE
GOING ON AND I'VE TALK ABOUT
BEFORE IS THE AREA OF THE
BESPOKE GENE THERAPY CONSORTIUM.
AN ACCELERATED MEDICINES
PARTNERSHIP WITH THE PARTNERSHIP
OF THE NIH AND A LARGER PROGRAM
IN THE SPACE WITH A VARIETY OF
INDUSTRY READERS, BIO TECH
LEADERS, PATIENT ORGANIZATIONS
AS WELL AS 11 DIFFERENT
INSTITUTES AND CENTERS AT THE
NIH.
AND WITH THIS PROGRAM WE'RE
LOOKING TO HAVE GENE THERAPIES
FOR EIGHT RARE DISEASES.
THAT'S SORT OF THE CEILING.
WE HOPE WE CAN MAKE THE CEILING
BUT THESE ARE COMPLICATED
PROGRAMS.
WE'RE GOING TO DO OUR BEST TO
GET TO EIGHT.
SIX OF THESE RARE DISEASES ARE
ONES NIH WILL BE PUSHING ON.
THE UPDATE I WANTED TO SHARE
WITH YOU TODAY IS THROUGH THE
BGTC WE SUBMITTED RARE PEDIATRIC
DISEASE DESIGNATIONS IN ORPHAN
DRUG DISEASE DESIGNATIONS AND
SIX OF OUR RRPDs WERE GRANTED BY
THE FDA AND THREE OF OUR ORPHAN
DRUG DESIGNATIONS GRANTED BY THE
FDA.
IN A MATTER OF ABOUT A PUBLIC
HEALTH.
THE FIRST APPROVAL WAS ABOUT A
MONTH AGO.
WE HOPE THE NEXT THREE ORPHAN
DRUG DESIGNATIONS WILL HAPPEN
RELATIVELY SOON AND WANT TO SAY
WHY THEY'RE IMPORTANT.
THESE ARE RARE DISEASES THAT ARE
VERY RARE.
WE HAVE TO DEMONSTRATE THE
PREVALENCE DATA ARE INDICATIVE
THESE ARE RARE DISEASES.
IF THE DRUG IS APPROVED THROUGH
RARE PEDIATRIC DISEASE
DESIGNATION IT'S ELIGIBLE FOR A
PRIORITY REVIEW VOUCHER.
THIS WAS NOT A GIVEN A FEW DAYS
AGO.
CONGRESS PASSED THE PRIORITY
REVIEW VOUCHER.
THIS WILL ALLOW IT TO BE SOLD
FOR PRICES THAT EXCEED OVER $100
MILLION.
THIS IS A BIG DEAL FOR THE
COMMUNITY.
THIS HELPS US DERISK WHAT WE'RE
DEVELOPING SO COMPANIES CAN TAKE
THEM UP IN A WAY THAT WILL
SUPPORT THEM IN THAT PROCESS.
THE ORPHAN DRUG DESIGNATION
SIMILARLY ENCOURAGES THE
DEVELOPMENT OF THE TREATMENT FOR
THE RARE DISEASES.
SO SEVEN YEARS OF MARKET
EXCLUSIVITY, TAX CREDITS AND
WAIVER OF FDA FEES.
AGAIN, THESE ARE REAL TANGIBLE
BENEFITS FOR DEVELOPING THESE
TREATMENTS AND SO WE'RE VERY
EXCITED TO BE CONTRIBUTING TO
THIS PROGRAM AND SO FAR THE
SUCCESS WE HAVE GIVES US EVEN
MORE CONFIDENCE AND EXCITEMENT
TO MOVE FORWARD IN THE BGTC
SPACE.
THIS IS A VILLAGE BUT I HAVE TO
SAY PG AND DOMINIQUE AND THE FDA
FOLKS ARE INCREDIBLE IN MOVING
THE FIELD FORWARD.
IT'S A BIG EFFORT FOR US ALL.
IT'S A VILLAGE.
I WANTED TO MAKE SURE EVERYONE
GETS CREDIT AS A BIG DEAL.
AND ANOTHER AREA I'VE SPOKEN ON
IS THE ACTIVE CLINICAL PROGRAMS
AND THE NATIONAL CLINICAL COHORT
COLLABORATIVE.
THESE TWO PROGRAMS HAVE SORT OF
MERGED INTO ONE AS THE ACTIVE IS
NOW CLOSING OUT, IT TOOK
ADVANTAGE OF THE FACT WE HAVE
THE N3C COHORT.
AND SO WHAT I WANTED TO SHARE IS
THE ACTIVE SIX CLINICAL TRIALS,
THERE WERE SEVEN ARMS IN THE
CLINICAL TRIAL AND ALL HAVE
COMPLETED ENROLLMENT, ON TIME,
UNDER BUDGET AND WE'RE VERY
EXCITED THAT THE FINAL CLINICAL
TRIAL HAS FINISHED THE METFORMIN
ARM OF THIS TRIAL FINISHED.
WE SHOULD BE SEEING TOP LINE
RESULTS FOR THE TRIAL FOR 28 DAY
COVID RESPONSE.
I THINK IT'S BY OCTOBER,
NOVEMBER IS WHEN WE EXPECT TO
SEE THE TOP LINE RESULTS AND
THEY'RE ALSO LOOKING AT LONG
COVID FOR THE PARTICULAR ARM.
THAT'S AN EXCITING FEATURE OF
THIS PARTICULAR ACTIVE TRIAL
WITH METFORMIN IS GOING A LITTLE
BIT LONGER.
THAT'S PARTLY THE REASON WE DID
THAT IS BECAUSE THERE WAS A
PRELIMINARY EVIDENCE IN THE
COVID CLINICAL TRIAL THAT SHOWED
METFORMIN SHOWED REDUCED
INCIDENTS OF MORE THAN 28 DAY
COVID RESPONSE IN LONG COVID.
SO THERE'S AN INKLING METFORMIN
MAY BE DOING SOMETHING.
IF THAT'S THE CASE WE WANTED TO
TEST IT IN A BIGGER TRIAL.
THAT'S WHAT WE DID.
WE ALSO TESTED THIS IN THE M3C
COHORT AND DID A TRIAL EMULATION
TRIAL AND WE CAN LOOK IN AT TH
COMPUTER TO LOOK AT THE OUTCOME
OF THE INDIVIDUALS IN ADDITION
TO THE RANDOMIZED TRIAL TO
COMPARE AND CONTRAST THE VALUE
OF THE EMULATION TRIALS AND WHAT
ARE WE MISSING AND ARE THERE
BIASES IN THE DATA WE SHOULD
LOOK FOR.
IT WILL HELP SET THE STAGE FOR
HOW TO USE ELECTRONIC HEALTH
RECORDS AND DATA SCIENCE TO
IMPACT HOW WE DESIGN CLINICAL
TRIALS IN THE FUTURE.
THAT'S ONE OF THE KEY VALUES
THAT WE'RE SEEING OUT OF THIS
PARTICULAR PROGRAM.
THE N3C USE OF THIS IS THROUGH
OUR FASTER PROGRAM AND LOOKING
AT WHETHER METFORMIN REDUCES THE
THREAT OF DEATH THROUGH LONG
COVID AND THE PAPER IS I THINK
FINALIZED AND I DON'T WANT TO
STEAL HER THUNDER.
IT REMAINS TO BE SEEN HOW IT
PLAYS OUT WITH THE RANDOMIZED
CLINICAL TRIAL.
STAY TUNED.
IT'S A GOOD EMERGING STORY.
THE RCT IS COMING SOON.
I WANT TO ROUND OUT HIGHLIGHTS
THAT HAVE HAPPENED OVER THE PAST
SUMMER WITH ANOTHER KEY
ENGAGEMENT AROUND COMMUNITY
ENGAGEMENT.
THERE'S AN EXTRACT ADVISORY
COMMITTEE AND THE PROJECT IS
THEY'RE ENGAGING THE PUBLIC IN
PARTNERS IN CLINICAL RESEARCH.
WE KNOW COMMUNITIES TO ENGAGE
THEM IT'S NOT A ONE SIZE FITS
ALL APPROACH.
THIS WILL TAKE A LOOK AT HOW NIH
IS MATURING FOR THOSE
PARTNERSHIPS.
I WORKED WITH A TEAM ACROSS THE
NIH AND NOW SARA DUNSMORE AND
DEVELOPED STUDIES IN THIS SPACE.
THESE WERE USED TO DEVELOP A
RESOURCE FOR PEOPLE ACROSS THE
NIH TO UNDERSTAND THE REALM OF
COMMUNITY ENGAGEMENT AND HOW
PEOPLE USED THEM BEFORE AND
DON'T HAVE TO REINVENT THE
WHEEL.
WE PUT THEM IN ONE AREA AND IT'S
PARTNERS IN RESEARCH DOT NIH
DOT-GOV AND GIVE YOU THE QR CODE
IF YOU'RE INTERESTED.
THESE SHOW FOR EXAMPLE THE TOOL
KIT FOR PATIENT FOCUSSED
DEVELOPMENT IN DRDRI HELPING
PATIENTS FIND RESEARCHERS
INVOLVED UP RARE DISEASES
THEY'RE INTERESTED IN.
WE HAVE FASTER TOGETHER WHICH IS
A TRIAL INNOVATIONS NETWORK
PRODUCT HELPING RESEARCHERS
UNDERSTAND HOW TO ENSURE THEY
PARTICIPATE IN CLINICAL TRIALS.
CEAL IS REPRESENTED IN THESE USE
CASES.
THESE ARE A VARIETY THING I
WANTED TO GIVE A FLAVOR FOR OF
WHAT WE TRY TO PULL TOGETHER IN
THE COMMUNITY.
THIS IS MEANT TO BE A DYNAMIC
SPACE.
SO IF YOU BOOKMARK THIS TAP
YOU'LL SEE THIS GROW OVER TIME.
LYRIC AND I DID A BLOG TO
HIGHLIGHT THESE USE CASES.
SO IF YOU'RE INTERESTED IN THAT
AS A BRIEF SUMMARY I SHOW THE QR
CODE THERE AS WELL.
ANOTHER ACTIVE WE'VE MENTIONED
IN THE PAST IS ON CARE FOR
HEALTH.
THIS WAS LAUNCHED EARLIER THIS
YEAR THROUGH THE COUNCIL OF
COUNCILS.
THIS IS A KEY PRIORITY FOR
DR. BERTAGNOLLI TO DEVELOP
NETWORK HUBS TO GET IN THE
PRIMARY CARE SETTINGS AND IN
COLLABORATION WITH CLINICAL
STRENGTHS WITHIN THE NIH.
PARTICIPANTS AND COMMUNITY
ENGAGEMENT IS A KEY PILLAR AND
WE WANT INNOVATIVE CLINICAL
TRIAL DESIGNS AND WANT TO
INCORPORATE PRIMARY CARE
COMMUNITIES.
THAT'S NOT GOING TO TO BE VERY
STRAIGHTFORWARD TO DO.
IT'S GOING TO BE A HEAVY LIFT.
I DON'T THINK THERE'S A MORE
IMPORTANT ACTIVITY WE COULD BE
DOING AT THE NIH.
I'M VERY EXCITED ABOUT MOVING
THIS PROJECT FORWARD I'M A
CO-CHAIR AND WE HAVE NIBCB AND
WE'RE THE CO-CHAIRS AND MIKE IS
THE DIVISION OF CLINICAL
INNOVATIONS AND HELPING GUIDE
EFFORTS AS WELL.
THE FIRST AWARDS FOR THE
PARTICULAR PROGRAM ARE COMING
OUT SOON.
I HOPE WITHIN THE FIRST 11 DAYS.
THAT WILL BE VERY IMPORTANT.
SO, I WANT TO ALSO END UP GOING
THROUGH THESE QUICKLY.
I KNOW YOU READ THE STRATEGIC
PLAN AND PROBABLY MEMORIZED IT.
I WON'T SPEND A LOT OF TIME ON
THIS BUT WANT TO END ON THIS
NOTE.
SO, THE STRATEGIC PLAN IS OUT.
AS YOU KNOW THIS WAS A LONG
PROCESS INCORPORATING A VARIETY
OF DISCUSSIONS THIS SLIDE IS
MEANT TO GIVE A SPECIAL SHOUT
OUT TO ALL OF YOU AND NCATS
STAFF AND MOST IMPORTANTLY THE
PEOPLE WHO DID THE BULK OF THE
WORK.
THEY HIT IT OUT OF THE PARK AND
COULDN'T BE MORE PROUD OF THE
STRATEGIC PLAN AND IF YOU
HAVEN'T TAKEN A LOOK, PLEASE DO
SO.
I'LL GO THROUGH A LITTLE BIT OF
WHAT IT LOOKS LIKE IN CASE YOU
HAVEN'T.
HERE ARE THE GOALS OF THE PLAN
AND IT MAY LOOK FAMILIAR TO YOU.
AUDACIOUS GOALS ARE MORE
TREATMENT FOR MORE PEOPLE
QUICKLY.
THOSE ARE THE FIRST THREE GOALS
BUT SET MORE STRATEGIC PLANNING
LIKE AND THEN ADDED A FEW MORE
GOALS.
THE FOURTH GOAL IS CROSS CUTTING
STRATEGIES TO ENHANCE POLICY
DEVELOPMENT AND DATA SCIENCE,
ETCETERA.
AND EFFECTIVE STEWARDSHIP OF
TRANSLATIONAL SCIENCE INTEGRITY
AND SOCIAL RESPONSIBILITY.
THOSE ARE OUR KEY FEATURES OF
WHAT WILL HAPPEN IN THE PLAN.
YOU'LL SEE OR PROBABLY EXPECT
MANY OF THESE GOALS ACTUALLY
OVERLAP WITH ONE ANOTHER.
AND THAT'S OKAY.
WE DON'T NECESSARILY NEED THEM
TO BE VERY DEFINED IN ONE
PARTICULAR GOAL.
IN FACT IF THEY'RE INTEGRIGRATEE
MAY BE ABLE TO LIFT MORE BOATS
AND ADVANCE DEVELOPMENT OF AN
ACCESS TO MORE TREATMENTS.
PARTICULARLY WITH THE UNMET
NEEDS.
HERE WE'RE FOCUSSING ON
DIAGNOSIS AND THERAPIES.
NEW MOLECULES AND TARGETS AND
CRITICAL ACTIVITIES WITH THE
CTSAs ARE SO IMPORTANT IN THE
WORK WE DO AND THE ACTIVITIES IN
PRECLINICAL TESTING.
FOR GOAL NUMBER 2 BENEFITING
FROM INCLUSIONARY SCIENCE AND
RESEARCH AND CLINICAL TRIALS AND
BRINGING COMMUNITIES AND
PATIENTS AS PARTNERS AND HEARING
VOICES LISTENING TO THOSE NEEDS.
ADDRESSING HEALTH DISPARITIES
AND USING IT ALSO TO HELP
SUPPORT A LARGER TRANSLATIONAL
SCIENCE WORKFORCE AND BREAKING
BARRIERS AND BOOSTING
EFFICIENCY.
EFFICIENCY IS A BIG DEAL FOR US
AT NCATS.
REALLY BREAKING DOWN THE
BARRIERS OF SCIENTIFIC
INNOVATIONS IS CRITICAL USING
DATA SCIENCE APPROACHES FOR
EXAMPLE, PLATFORM TECHNOLOGIES
AND PREDICTIVE MODELS.
THERE'S ALSO KEY OPERATIONAL
PIECES WE DO.
HOW WE WORK WITHIN NCATS IS
IMPORTANT AND MODERNIZING THOSE
AND ENHANCING THOSE FOR MORE
EFFICIENT ACTIVITIES ARE KEY
PIECES AS WELL.
GOAL NUMBER FOUR IS THE CROSS
CUTTING TEAMS.
TEAM SCIENCE, PARTNERSHIPS,
COLLABORATIONS, PUBLIC-PRIVATE
PARTNERSHIPS AND DATA SCIENCE
STANDARD AND SUPPORTING THE WORK
WE DO AND DEVELOPING PRINCIPLES
AND USE THOSE TO SUPPORT THE
SCIENCE WE DO AS WELL.
AND GOAL 5 IS CHAMPIONING THE
EFFECTIVE STEWARDSHIP OF SEENS
THROUGH TRANSPARENCY.
OPEN AND TRANSPARENT DATA
ACCESS, REPRODUCIBLE AND ETHICAL
RESEARCH.
DEVELOPING POLICIES AND
GUIDANCES FOR THE WORK WE DO AND
DEVELOPING THAT INFRASTRUCTURE
FOR IMPACT.
I ALWAYS SAY THAT A LOT OF THE
WORK WE DO IS SUPPORTING DEEP
INFRASTRUCTURE AND WITHOUT THAT
DEEP INFRASTRUCTURE WE DO NOT
HAVE A CHANCE OF BUILDING UPON
IN TERMS OF THE INNOVATIONS WE
WANT TO HAVE.
SO INFRASTRUCTURE IS INCREDIBLY
IMPORTANT.
SO THESE ARE THE KEY FEATURES OF
OUR ACTIVITIES WITHIN THE
STRATEGIC PLAN.
AND I THINK -- I SET A SIMILAR
VERSION OF THIS YESTERDAY BUT
WANT TO -- I LIKED IT SO MUCH I
WANT TO SAY IT TODAY BECAUSE I
FEEL STRONGLY ABOUT THIS.
AS MUCH WE FOCUS ON THE PROJECT,
PROTOCOLS IT'S THE PEOPLE WHO
MAKE IT POSSIBLE FROM PUSHING
THE BOUNDARIES TO THOSE WITH
LIVED EXPERIENCES WHO STAND TO
BENEFIT FROM OUR COLLECTIVE WORK
AND ALSO BY LISTENING TO THEM
AND LEARNING FROM THEM.
TO THE PATIENT SCIENTISTS
INVOLVED IN THE WORK WE SUPPORT
AND TO ALL OF OUR PUBLIC AND
PRIVATE SECTOR PARTNERS IN THESE
ACTIVITIES.
YOU ALL ARE THE HEARTBEAT OF
WHAT NCATS DOES.
AT THE END OF THE DAY IT'S NOT
JUST ABOUT ADVANCING THE SCIENCE
WE DO BUT ADVANCING THE LIVES
WE'RE HERE TO HELP.
AND PEOPLE DRIVE THE PURPOSE.
I FEEL STRONGLY THAT THE
STRATEGIC PLAN IS A REFLECTION
OF THAT SENTIMENT.
SO I WANTED TO RELAY THAT HERE
TODAY.
WHAT'S NEXT FOR OUR STRATEGIC
PLAN?
WE'RE NOT OVER.
OUR NEXT PLANS ARE TO HAVE A
SERIES OF ROUNDTABLES TO DISCUSS
WHAT TO DO NEXT AS A COMMUNITY.
AND SO -- THOSE WILL BE
HAPPENING OVER THE COURSE AT THE
END OF THE MONTH AND EARLY
OCTOBER.
IF YOU HAVEN'T HAD A CHANCE TO
REGISTER, PLEASE DO.
SO THAT'S MY PRESENTATION IN A
NUTSHELL TODAY AND JUST TO
SUMMARIZE AGAIN THE ACTIVITIES
WE'VE GONE OVER ARE SHOWN HERE.
I DON'T NEED TO GO THROUGH IT
BUT ALSO WANT TO RE-HIGHLIGHT WE
HAVE A FEW OTHER TOPICS
DISCUSSED TODAY FROM JANINE AND
SHARI AND MIKE AND CHRISTINE SO
STAY TUNED FOR THOSE EVERY SO
OFTEN WE RECEIVE LETTERS AND WE
RECEIVED SUCH A LETTER TODAY AND
EARLIER THIS WEEK.
THIS LETTER CAME FROM OUR NCATS
COUNCIL I'M PRESENTING A LETTER
FROM THE COUNCIL TO THE COUNCIL
WHICH FEELS A LITTLE ODD.
IT ALSO SOUNDS OMINOUS BUT IT'S
NOT.
IT'S QUITE THE OPPOSITE.
IT'S VERY INSPIRING.
IT'S A FORMAL REQUEST TO
INITIATE THE PROCESS OF A
TRANSITION OF NCATS TO AN
INSTITUTE.
THIS CAME UP VERY BRIEFLY AT OUR
LAST COUNCIL GATHERING AND SO
THE COUNCIL GOT TOGETHER TO
WRITE AND SIGN THIS LETTER.
AND I WANT TO BRING A COUPLE
THINGS TO YOUR ATTENTION ABOUT
THIS.
FIRST OF ALL THE LETTER HAS BEEN
SIGNED BY ALL COUNCIL MEMBERS
PRESENT AND ALSO MANY COUNCIL
MEMBERS PAST.
AND SO VERY HEARTWARMING TO HAVE
SUCH A ROBUST RESPONSE.
IT PRESENTS A COMPELLING SET OF
ARGUMENTS THAT ARE BASED ON
PRECEDENT AND AS TO WHY NCATS
SHOULD BECOME AN INSTITUTE AND
OTHER CURRENT INSTITUTES THAT
HAVE GONE THROUGH THE PROCESS AS
WELL ARE HIGHLIGHTED.
NHGRI AND NIMHD AND NINR HAVE
GONE THROUGH IT.
I'M IMPRESSED BY THE
THOUGHTFULNESS IN THE LETTER AND
THE FACT IT'S BUILT ON
PRECEDENCE.
BEFORE I ASK ANNIE KENNEDY TO
SAY A FEW WORDS ABOUT THIS
BECAUSE SHE WAS THE ONE WHO SENT
IT TO ME SO I GUESS SHE'S ON THE
HOOK.
I WANT TO SAY HOW HUMBLED I AM
WITH THIS OVERWHELMING SUPPORT
FROM OUR COUNCIL.
IT'S AN IMMENSE HONOR TO LEAD
NCATS AND WOULD BE AN HONOR TO
LEAD NCATS TO ACCOMPLISH MORE
AND WITH THAT BECAUSE WE'RE IN
THE FIRST QUARTER I KNOW MANY OF
US ARE STRUGGLING AND DRAINED
AND WANT TO READ JUST ONE
PARAGR
PARAGRAPH.
THE NCATS ADVISORY COMMITTEE
APPLAUDS THE LEADERSHIP FOR YOUR
SCIENCE AND UNWAVERING TO YOUR
COMMITMENT TO DELIVERING MORE
TREATMENTS FOR ALL PEOPLE MORE
QUICKLY.
AS AN ENTITY, YOU ARE
UNSURPASSED IN INNOVATION,
COLLABORATIVE SCIENCE AND
STIMULATING BIOMEDICAL RESEARCH,
THERAPY DEVELOPMENT AND
IMPACTFUL HEALTH OUTCOMES.
SO, THANK YOU TO OUR COUNCIL FOR
THESE WONDERFUL WORDS.
THEY'RE INSPIRING.
SO, WITH THAT I WANT TO TAKE A
MOMENT TO THANK YOU ALL AND ASK
FOR ANY QUESTIONS AND WOULD ALSO
LIKE TO GIVE ANNIE KENNEDY A
MOMENT TO RESPOND TO THE
COMMENTS AND MAYBE WE'LL DO THAT
FIRST BUT THANK YOU SEARCH.
>> LET'S LET AMY GO FIRST IF
THAT'S OKAY.
>> FIRST, THANK YOU VERY MUCH
DR. RUTTER FOR THE DIRECTOR'S
REPORT WHICH I THINK SPEAKS TO
WHY THE COUNCIL FELT IT WAS TIME
TO BRING THIS FORWARD.
AS NCATS BEGINS TO IMPLEMENT THE
THOUGHTFUL AND COMPREHENSIVE
STRATEGIC PLANS YOU JUST
PRESENTED TO US AND TO THE
COMMUNITY, WE FELT THAT IT WAS
REALLY IMPORTANT THAT IN ORDER
TO ENABLE NCATS TO DELIVER MORE
TREATMENTS FOR ALL PEOPLE MORE
QUICKLY, THAT OUR ADVISORY
COUNCIL CONSIDER THE
UNPARALLELED WORK AND LEADERSHIP
OF NCATS AS WELL AS PAST
PRECEDENCE YOU JUST OUTLINED
FROM OTHER NIH INSTITUTES AS
THEY EVOLVED WHEN THEY HAD
INITIALLY BEEN IMPLEMENTED AND
BRING STRONGLY THAT THE
IMPLEMENTATION OF NCATS MISSION
GOING FORWARD WILL BEST BE
ACHIEVED FOR NCATS TO BE
ELEVATED FROM CENTER TO AN
INSTITUTE.
I'VE TAKEN NOTES THIS MORNING
FOR YOUR DIRECTOR'S REPORT AND
FROM SOME OF THE PRESENTATIONS
WE'VE BEEN HEARING AND NCATS
UNIQUELY STANDS AT A CROSSROADS
OF SCIENCE.
NCATS LEADERSHIP IS
UNPARALLELED.
SOME IMPACT PORTFOLIOS WITHIN
NIH YOU LEAD ARE ADVANCING
A.I./M.L., QUANTUM COMPUTING,
HIGH THROUGHPUT CRISPR AND
TRANS-NIH COLLABORATIONS,
SPURRING INNOVATION AND
EXPANDING THE DRUG LANDSCAPE AND
RESEARCH INNOVATION, LEADING THE
NATION'S LARGEST CLINICAL AND
TRANSLATIONAL SCIENCE
INFRASTRUCTURE AND SO MUCH MORE.
WE BELIEVE IT'S TIME TO INITIATE
THE PROCESS AND FEEL STRONGLY
THERE'S COMPELLING EVIDENCE THAT
NCATS' MIRRORS PAST PRECEDENT
WITH THE POTENTIAL OPPORTUNITIES
THAT EXIST WITHIN DESIGNATION OF
AN INSTITUTE.
IN DOING SOME OF THE RESEARCH TO
BRING THIS LETTER FORWARD WE
WENT THROUGH THE CONGRESSIONAL
RECORDS THAT LED TO THE
EVOLUTION OF SOME OF THOSE
INSTITUTES YOU NAMED.
AND I'D LIKE TO READ ONE OF THE
QUOTES FROM THE CONGRESSIONAL
RECORDS.
IF NCATS CAN OPERATE UNDER THE
STATEMENT LEGISLATIVE
AUTHORITIES AS OTHER NIH
RESEARCH INSTITUTES, END QUOTE,
SUCH AS CONDUCTING TRIALS BEYOND
PHASE II B WE BELIEVE IT WOULD
BOOST NCATS FUTURE AND UNLOCK
ITS FULL CAPACITY TO DELIVER
MORE TREATMENTS FOR ALL PEOPLE
MORE QUICKLY.
SO WE THANK YOU FOR YOUR WORK
AND FEEL STRONGLY AND STAND
READY TO SUPPORT YOU IN THIS
ENDEAVOR.
>> SO WITH THAT LETTER THIS IS
AN IMPORTANT STEP FOR THIS TO
HAPPEN.
BUT THERE ARE MANY STEPS THAT
WILL BE REQUIRED TO MAKE IT
HAPPEN.
MANY KNOW THIS WOULD BE A
CONGRESSIONAL CHANGE AND SO THE
PATH TO GET THERE CAN BE VARIED.
WE'LL DO WHAT WE CAN TO MOVE IT
FORWARD INTERNALLY AND SEE WHAT
WE CAN DO WITH THAT.
THIS PROVIDES US WITH THE VERY
STRONG LANGUAGE TO HELP SUPPORT
THAT AND SO IT IS REALLY A BIG
STEP SO THANK YOU VERY MUCH FOR
YOUR CONFIDENCE IN NCATS AND IN
OUR ABILITY TO MOVE TO AN
INSTITUTE.
IT'S EXCITING TO THINK ABOUT.
I WILL SAY THAT THIS LETTER IN
ITS ENTIRETY WILL BE PUCK LIB ON
OUR WEBSITE SO WITH THAT I'D
LIKE TO OPEN IT UP TO ANY OTHER
QUESTIONS RELATED TO THAT OR ANY
OTHER COMMENTS YOU MIGHT HAVE.
IF NONE, THAT'S OKAY TOO.
ALL RIGHT.
VERY GOOD.
IN THAT CASE, SHALL WE MOVE ON?
OH, IS IT LUNCH NEXT?
OKAY.
COULDN'T HEAR.
THANK YOU.
SO YES, WE SHOULD MOVE ON.
PLEASE, HAVE LUNCH AND WE'LL BE
BACK AT WHAT TIME?
1:10.
SO THANK YOU.
WE'LL SEE YOU SOON.
ALL RIGHT, I HOPE EVERYONE HAD A
GOOD LUNCH.
WELCOME BACK.
WE ARE GOING TO START OUR NEXT
SEGMENT FOR THE PRESENTATIONS
MOVING FORWARD.
FIRST UP AGAIN VERY EXCITED
ABOUT THIS PRESENTATION AND I'LL
TURN IT OVER TO JEANITA.
>> AWESOME.
GOOD AFTERNOON, EVERYONE.
I HAVE THE PLEASURE OF KICKING
OFF TODAY'S AFTERNOON SESSION
AFTER LUNCH SO HOPEFULLY YOU'RE
FEELING ENERGIZED AND HAD YOUR
SECOND CUP OF COFFEE AND HERE TO
TALK ABOUT THE EFFORTS IN THE
SPACE OF DIVERSITY, EQUITY AND
INCLUSION AND ACCESSIBILITY IN
NCATS AND BEYOND.
I'LL KICK OFF THE CONVERSATIONS
AND HAND IT OVER TO MY DEAR
COLLEAGUE TO TALK ABOUT SPECIFIC
WORK SHE'S DOING IN THE
PRECLINICAL SPACE.
TO GIVE A QUICK OVERVIEW OF WHAT
WE'LL BE DIVING INTO IN THE
CONVERSATION WE'LL BE EXPLORING
THE DIFFERENT STRATEGIC
INITIATIVES WE'VE BEEN UTILIZING
TO NOT ONLY CULTIVATE A DIVERSE
WORKFORCE BUT FOSTERING
INCLUSIVE LEADERSHIP AND
THINKING OF WAYS TO INTEGRATE
EQUITY IN OUR RESEARCH PROGRAMS
AND DEVELOPING IMPACTFUL AND
SUSTAINABILITY DEIA PROGRAMS AT
NCATS.
SO EARLIER WE TALKED ABOUT THE
STRATEGIC PLAN AND YOU CAN SEE
AN OUTLINE OF NOT ONLY OUR GOALS
OF MORE TREATMENTS FOR ALL
PEOPLE MORE QUICKLY BUT A NOD IT
OUR TRANSLATIONAL SCIENCE
PRINCIPLES AND GUIDES US IN WAYS
TO APPROACH THE WORK TO OVERCOME
LONG STANDING CHALLENGES WITH
TRANSLATIONAL RESEARCH PIPELINE.
YOU CAN SEE THE EIGHT PRINCIPLES
DEVELOPED.
AND MANY LIKELY SEEM FAMILIAR TO
YOU, TEAM SCIENCE, BOUNDARY
CROSSING PARTNERSHIPS AND
CROSS-CUTTING SOLUTIONS BUT IF
YOU LOOK TO THE FAR RIGHT OF THE
SCREEN YOU SEE ONE AROUND
ADVANCING DIVERSITY, EQUITY,
INCLUSION AND ACCESSIBILITY IN
RESEARCH.
THAT'S GOING TO BE THE CRUX IN
HOW WE'RE DOING THAT AND WHAT'S
THAT LOOK LIKE AND HOW DOES DEIA
IN A SCIENTIFIC DOMAIN LOOK
LIKE.
IT GOES BEYOND JUST LOOKING AT
OUR WORKFORCE AND ENSURING THAT
IS REPRESENTATIVE AND REFLECTIVE
OF THE COMMUNITIES WE SERVE AND
THINKING ABOUT INCLUDING DIVERSE
PERSPECTIVES ALONG OUR RESEARCH
PROCESS TO ENSURE THOUGHTS AND
VALUES ARE HEARD.
IT LOOKS LIKE ENSURING A
WORKFORCE INCLUSIVE WHERE PEOPLE
FEEL THEY BELONG AND CREATING
SPACE FOR EXTERNAL COMMUNITY
PARTNERS KNOW HOW TO ACCESS
RESOURCES AND OPPORTUNITIES.
IT LOOKS LIKE ENSURING WE HAVE
EQUITY IN OUR PROCESSES AND
MAKING SURE WE ARE CREATING
SPACES THAT ARE FAIR FOR ALL
PEOPLE TO THRIVE.
I WANTED TO TALK ABOUT MY ROLE
AS THE INAUGURAL CHIEF DIVERSITY
OFFICER FOR MANY IT MAY BE A NEW
ROLE TO HELP UNDERSTAND WHAT IT
IS I DO IN THIS SPACE.
SO YOU CAN SEE ON THE SLIDE HERE
A NUMBER OF DIFFERENT THINGS BUT
REALLY WHEN IT COMES DOWN TO IT,
MY ROLE IS TO FOCUS ON WAYS IN
WHICH I CAN HARMONIZE, ELEVATE
AND ALIGN VARIOUS DEIA EFFORTS
HAPPENING NOT ONLY AT NCATS BUT
TO NIH AT LARGE.
I DO THIS BY SERVING AS A
THOUGHT PARTNER AND CONNECTING
WITH DIVISIONS AND OFFICES,
HELPING TO BRAIN STORM IN WAYS
IN WHICH WE CAN INTEGRATE
VARIOUS PRINCIPLES INTO THE WORK
STREAMS.
LEVERAGING DATA TO HELP INFORM
THE DIFFERENT INITIATIVES WE'RE
PUTTING FORTH AND THINKING ABOUT
WAYS IN WHICH WE CAN GET IN THE
COMMUNITY AND PROMOTE OURSELVES
AS AN EMPLOYEE OR AGENCY OF
CHOICE FOR COLLABORATION AND FOR
POTENTIAL WORKFORCE NEEDS.
SO, ON THE NEXT SLIDE WE'LL TALK
ABOUT THE DIFFERENT STRATEGIES
WE'VE BEEN DOING HOLISTICALLY
ACROSS THE BOARD THINKING ABOUT
DIVERSITY, EQUITY AND INCLUSION
AND ACCESSIBILITY.
I LOVE THIS SLIDE BECAUSE FOR ME
IT HELPS TAKE A STEP BACK AND
THINK BROADLY ABOUT ALL THE
DIFFERENT PLACES THIS WORK
INTERSECTS.
MOST PEOPLE WHEN THEY HEAR DEIA
THINK ABOUT RECRUITMENT, WHICH
IS A PART OF THE PRINCIPLES.
BUT DEIA EXPANDS BEYOND OUR
WORKFORCE AND RESOURCES,
RESEARCH, THE WAY WE ENGAGE WITH
EXTERNAL COMMUNITIES.
THE SLIDE DEMONSTRATE WHAT I
CALL BRIGHT IDEAS, DIVERSION,
INCLUSION AND EQUITY STRATEGIES.
YOU CAN SEE ON THE SLIDE THERE'S
A GAMUT OF DIFFERENT ACTIVITIES.
WE'LL WALK THROUGH MANY
THROUGHOUT THE CONVERSATION BUT
I WANT TO HIGHLIGHT A FEW.
YES, WE'RE GOING TO BE TALKING
ABOUT SOME OF OUR OUTREACH AND
ENGAGEMENT STRATEGIES AND
RECRUITMENT EFFORTS AND THINKING
ONCE WE GET PEOPLE IN THE FOLD,
WHAT ARE WE DOING TO GROW THEM
INTO LEADERS OF THE FUTURE.
THINKING ABOUT OUR DISPARITY
RESEARCH WE'RE LEADING AND HOW
WE'RE ENGAGING WITH THE PATIENT
GROUPS AND COMMUNITY
ORGANIZATIONS.
TO START TO GET PEOPLE IN THE
FOLD AND IN OUR WORKFORCE WE
WANT TO REACH OUT AND CONNECT.
WE'VE DONE A HUGE LIFT OUR
RESEARCH AND GET OUT IN THE
COMMUNITY AND YOU SEE A FEW I
WANT TO POINT OUT.
THESE ARE UNTAPPED AREAS WE'VE
BEEN REACHING OUT TO MAKE SURE
WE'RE POSITIONING NCATS AS A
MODEL EMPLOYER FOR FUTURE
EMPLOYEES OR STAFF MEMBERS.
YOU CAN SEE RECENTLY WE'VE DONE
ENGAGEMENTS THE PRIDE FESTIVAL
AND AT GALLAUDET UNIVERSITY
TAPPING INTO THE DEAF OR HARD OF
HEARING COMMUNITY AND SERVING AS
THOUGHT LEADERS IN THE DIVERSITY
SPACE, CONNECTING WITH VARIOUS
CHEMISTS AND CONNECTING WITH
DIFFERENT VENUES SUCH AS THE
NATIONAL URBAN LEAGUE OR THE
CAREER FAIR.
THERE'S INITIATIVES AND GETTING
THE NEXT GENERATION EXCITED
ABOUT TRANSLATIONAL SCIENCE.
SO WE'VE STARTED A K-12 OUTREACH
AND ENGAGEMENT PROGRAM THAT'S
ALLOWED US TO GET IN THE
COMMUNITY AND SHOW PEOPLE IT'S
OKAY TO FAIL WHILE DOING
RESEARCH AND GET THEM EXCITED
ABOUT THE IDEA OF TEAM SCIENCE
AND COLLABORATING WITH ONE
ANOTHER.
BEYOND RECRUITMENT WE WANT TO
LEVERAGE DATA TO UNDERSTAND HOW
WE'RE DOING.
AND LOOKING AT DATA ASSOCIATED
WITH THE WORKFORCE AND YOU SEE
THE RACE AND ETHNICITY DATA AND
SETS AND DISABILITY DATA FOR
STAFF.
THIS IS UPDATED FROM THE OFFICE
OF EQUITY, DIVERSITY AND
INCLUSION AND LOOK AT
INFORMATION FROM SURVEYS AND
FOCUS GROUPS TO UNDERSTAND HOW
WE'RE DOING ONCE WE UNDERSTAND
HOW OUR STAFF IS HERE.
WE'RE WORKING DILIGENTLY TO
DEVELOP A RECRUITMENT AND HIRING
TOOLKIT TO MAKE SURE WE'RE
BAKING IN EQUITY THROUGHOUT THE
PROCESSES AND EXPANDING THE POOL
OF POTENTIAL APPLICANTS.
AND LEVERAGING THINGS LIKE AND
HOW THEY PERCEIVE WE'RE DOING
AND IF IT'S AN ORGANIZATION OF
CHOICE AND HOW WE'RE DOING IN
THE IMPLEMENTATIONEST.
AND ALL THE SEMINARS WE LISTED
AND I'LL POINT TO WHAT THEY'RE
REPRESENTING ARE BASED OFF
FEEDBACK WE SAW IN THINGS LIKE
OUR FEDERAL EMPLOYEE VIEW POINT
SURVEY AND THINGS WE'RE SEEING
FROM OUR STAFF OF POTENTIAL
OPPORTUNITIES AND TRAIN TO THE
TRAINERS SESSIONS IN MIC
MICROAGGRESSIONS TO GETTING
STAFF COMFORTABLE AND CONTENT ON
THE TOPIC.
WE PARTNERED WITH THE DIVERSITY,
EQUITY AND INCLUSION OFFICE TO
EXPLORE ACCESSIBILITY REALIZING
AN AREA OF OPPORTUNITY TO
UNDERSTAND HOW TO IMPLEMENT
COMPLIANCE INTO YOUR WORK
PRODUCTS AND DIGITAL
ACCESSIBILITY.
ON THE FAR RIGHT YOU SEE SOME OF
THE GROUND WORK WE'VE BEEN DOING
AND HELPING PEOPLE UNDERSTAND
SYSTEMIC BARRIERS THAT HAVE
CHALLENGED US IN THE SPACE AND
HELPING PEOPLE GROW AND
UNDERSTAND AND BECOMING MORE
ENGAGED EMPLOYEES.
COUNCIL IS MADE UP OF
REPRESENTATION FOCUSSED ON
INITIATIVES TO ADVANCE DEIA
THROUGH VARIOUS WORKING GROUPS
AND WE HAVE OUR EMPLOYEE
ENGAGEMENT COMMITTEE FOCUSSED ON
CREATING POSITIVE WORK BY
CULTURE THROUGH A VARIETY OF
ACTIVITIES LIKE NCATS
ASSOCIATION WHICH IS COMING UP
SOON AND PROFESSIONAL
DEVELOPMENT THROUGH OUR BACK
CLUBS AND SEMINAR SERIES.
AND THE ASSISTANT COMMITTEE
FOCUSSED ON FOSTERING INCLUSIVE
WORKPLACE FOR MEMBERS TO BRAIN
STORM ON IDEAS AND COME UP WITH
COLLABORATIVE SOLUTIONS FOR
THEIR WORK PROFILES.
AND THE LAST PIECE I WANTED TO
HIGHLIGHT ARE SOME OF THE THINGS
WE'RE DOING IN CONTRIBUTIONS TO
THE LITERATURE.
FROM VARIOUS STAFF MEMBERS WE
HAD JOURNAL ARTICLES PUBLISHED.
.
OPINION PIECES AND AN ARTICLE
THAT HIGHLIGHTS STRATEGIES
AROUND WAYS IN WHICH TO
INTEGRATE DEIA IN THE
TRANSLATIONAL SCIENCE SPECTRUM
AND A MORE RECENT -- WELL, END
OF LAST YEAR AN ARTICLE THAT WAS
AUTHORED EXPLORING SICKLE CELL
DISEASE AND IMPLICATION AROUND
THAT DISEASE AND FUTURE
DISEASES.
YOU CAN SEE WE'RE APPROACHING
DEIA FROM SO MANY FACETS IN THE
ORGANIZATION.
AND SO I'VE GIVEN YOU A SNAPSHOT
OF WHERE WE'VE BEEN AND WHAT
WE'RE DOING AND NOW I WANT TO
TALK ABOUT WHERE WE'RE GOING.
IN ORDER TO BE SUSTAINABLE IN
THE SPACE IT'S IMPORTANT TO
LEVERAGE THE OUTPUT FROM THE
STRATEGIC PLAN AS A LAUNCHING
PAD FOR WHAT WE'LL BE DOING IN
THIS DEIA REALM.
SO OVER THE LAST YEAR WE SPENT A
LOT OF TIME GATHERING
INFORMATION EXPLORING DIFFERENT
DATA SOURCES EMPOWERING OUR
LEADERS AND SUPERVISORS TO BE
EQUIPPED IN THE SPACE AND NOW
POSITIONED TO DEVELOP A ROAD MAP
THAT'S GOING TO OUTLINE THINGS
WE'RE DOING IN THIS SPACE THAT
WILL SPECIFICALLY FOCUS ON OUR
ORGANIZATIONAL CAN CULTURE AND
RELATING THAT TO OUR DEIA
EFFORTS.
IN THE ROAD MAP WHICH IS AN
EARLY OUTPUT OF OUR STRATEGIC
PLANS WE'LL OUTLINE PILLARS AND
OBJECTIVES WE'LL FOCUS ON IN THE
NEXT COUPLE YEARS AS WELL AS
STRATEGIES AND ACTIONS WE CAN
USE DURING THE IMPLEMENTATION
PHASE.
WE GATHERED DATA FROM DIFFERENT
SOURCES.
I'VE BEEN ON BOARD ALMOST A
YEAR.
I'VE HAD MORE THAN 95 MEET AND
GREETS, CONVERSATIONS, REALLY
TRYING TO UNDERSTAND THE
LANDSCAPE WE'VE HAD ASSESSMENTS
DONE AND SURVEY DATA AND HAVE
BEEN ABLE TO COME UP WITH
PROPOSED PILLARS FOR OUR ROAD
MAP.
KEEPING IN MIND OUR NORTH STAR
OF THAT MORE TREATMENTS FOR MORE
PEOPLE MORE QUICKLY.
ON THE SCREEN YOU CAN SEE OUR
FOUR COMPOSED PILLARS FOR OUR
ROAD MAP.
YOU CAN SEE AGAIN THEY FOCUS NOT
ONLY ON OUR WORKFORCE BUT
EVERYTHING WE DO.
THE FIRST PILLAR IS AROUND
CULTIVATING A DIVERSE AND
INCLUSIVE AND REPRESENTATIVE
WORKFORCE TO ACHIEVE THE NCATS
MISSION.
IN THE SECOND PILLAR WE'RE GOING
BEYOND GETTING PEOPLE HERE BUT
THINKING HOW TO CREATE AN
ENVIRONMENT THAT'S SAFE AND
WELCOMING SO PEOPLE KNOW THEY
BELONG AND HAVE THE ABILITY TO
GROW WITHIN THE ORGANIZATION.
THE THIRD PILLAR IS FOCUSSING ON
OUR RESEARCH.
WE'LL HEAR A LOT ABOUT THE
EFFORTS WE'RE DOING IN THE
RESEARCH SHORTLY BUT HERE WE'RE
LOOKING TO INVEST AND SUPPORT
RESEARCH AREAS AND INITIATIVES
THAT REDUCE HEALTH INEQUITIES
AND THE FINAL PILLAR IS ON
RESOURCES FUNDING OPPORTUNITIES
AND DATABASES AND ENSURING USING
THEM TO ENGAGE COMMUNITIES,
RAISE AWARENESS AND SUPPORT
INNOVATION ACROSS OUR ENTIRE
TRANSLATIONAL SCIENCE SPECTRUM.
SO WE'VE BEEN REALLY EXCITED
WITH THE ENGAGEMENT WE'VE BEEN
ABLE TO HAVE OVER THE LAST FEW
MONTHS WITH STAFF AS WE'RE
BUILDING, CO-CREATE THE ROAD MAP
AND OBJECTIVES AND PILLARS THAT
WILL BE REPRESENTED.
SO WE'RE GOING TO BE PUTTING
THAT OUT BY END OF FIRST QUARTER
OF FY25.
SO STAY TUNED.
THE LAST THING I WANT TO TALK
ABOUT BEFORE HANDING IT OVER TO
MY COUNTERPART IS TO TALK A
LITTLE BIT ABOUT ONE OTHER THING
YOU'LL FIND IN THE STRATEGIC
PLAN.
WE NOW HAVE CORE VALUES IN THE
STRATEGIC PLAN.
THEY HELP AND HOW TO ENGAGE WITH
EXTERNAL PARTNERS.
I LOVE THE GRAPHIC BECAUSE IT
HELPS CONNECT ALL THE WORK IN
THE STRATEGIC PLAN AND WILL HELP
DRIVE US AS WE'RE DEVELOPING THE
ROAD MAP YOU CAN SEE THE TEAM
SCIENCE AND COLLABORATION AND
INNOVATION AND CREATIVITY AND
GENERALIZABLE SOLUTIONS AND
INTEGRITY AND DIVERSITY,
INCLUSION AND ACCESSIBILITY AND
LOCK
OUR VISION.
AS I GET READY TO HAND IT OVER
I'LL CLOSE IT UP WITH ONE OF MY
FAVORITE SLIDES BECAUSE THE
BRINGS TOGETHER THE IDEAS OF
DIVERSITY, EQUITY, INCLUSION,
ACCESSIBILITY AND HOW THEY CAN
LEAD TO BELONGING BUT TALKING
ABOUT EVERYTHING THAT IS GOING
TO BE NECESSARY TO BE
SUSTAINABILITY.
IT'S GOING TO TAKE US --
CONTINUE TO HAVE CONVERSATIONS
LIKE THIS AND INTEGRATING IT
INTO OUR ORGANIZATIONAL FABRIC.
HAVING LEADERS WILLING TO LEAD
BY EXAMPLE AND BE TRANSPARENT
ALONG THE WAY AND PRIMARILY ONE
OF THE BIGGEST THINGS IS GRACE
AS WE'RE GOING THROUGH THIS
SPACE.
WE NEED TO LEND EACH OTHER GRACE
TO REALLY BE WORKING TOWARDS
CREATING THE SPACE WHERE WE HAVE
COMMUNITY AND BELONGING.
SO I HOPE AS I'VE SET THE STAGE
FOR SOME OF THE VARIOUS THINGS
WE'VE BEEN DOING IN DEIA ACROSS
NCATS I'VE HELPED TO OPEN YOUR
APERTURE FOR LOOKING AT IT AS A
THING AS A WAY OF THINKING AND
IT TOUCHES EVERYTHING WE DO
WITHIN OUR ORGANIZATION.
WITH THAT I LOOK FORWARD TO
QUESTIONS YOU MIGHT HAVE BUT
HAND IT OVER TO DR. HAUGABOOK
FOR HER PRESENTATION ABOUT WHAT
SHE'S DOING IN THE PRECLINICAL
SPACE.
THANK YOU SO MUCH.
>> I'M DR. SHARRIE HAUGABOOK AND
THE ACTING HEAD OVER SCIENTIFIC
MANAGEMENT GROUP IN THE
THERAPEUTIC DEVELOPMENT BRANCH.
MAY I JUST ASK FOR MY NOTES,
PLEASE?
>> THANK YOU.
FIRST A PARALLEL FOR BIO MEDICAL
LEADER RESEARCH PERSPECTIVE.
PROVIDE A BROAD OVERVIEW IN THE
BIOMEDICAL RESEARCH ENTERPRISE
AND WHY ADVANCING EQUITY AT THAT
STAGE WE CONDUCT RESEARCH
EQUALLY IMPORTANT FOLLOWED BY
WHAT WHAT WE'RE DOING.
FIRST LET'S WHAT IS DEPICTED.
IT SHOWS A BASEBALL GAME WITH
ONLOOKERS OF DIFFERENT TYPES
PEERING OVER A FENCE.
THE SCENERY CHANGES FROM REALITY
TO EQUALITY TO EQUITY AND THEN
JUSTICE AS THE BOXES DIFFER.
ADVANCING EQUITY LEADS TO
JUSTICE.
THAT'S WHEN ALL THE SUPPORTS ARE
THERE AND YOU DON'T NEED THAT TO
SEE THE GAME IN THE CASE.
IN THE CONTEXT OF BIOMEDICAL
RESEARCH THERE'S A SIMILAR
PERSPECTIVE.
IN THE ADAPTATION THE DIFFERENT
COLORS REPRESENT THE FOUNDATION
OF SCIENTIFIC EFFECTIVENESS AND
PROGRESS.
THEY ARE RESEARCH CAPACITY AND S
AND PRESTIGE OR PRIVILEGE OR
YELLOW AND OPEN SCIENCE
PRACTICES IN GREEN.
THE BOX REPRESENT ACCESS AND
AVAILABILITY TO THE RESOURCE AND
DISTRIBUTION VARIED BY HIGH,
MIDDLE OR LOW RESOURCE
INSTITUTIONS.
THE IS WHERE EFFECTIVENESS MEANS
SOCIETAL.
FOR US THAT'S IN THE PUBLIC
HEALTH AND COMES AS A RESULT OF
RESEARCH FINDINGS AND BEING ABLE
TO MAKE RESEARCH OUTPUT
AVAILABLE TO OTHERS INCLUDING
THOSE FOR WHOM THE RESEARCH IS
CONDUCTED.
ADDING TO THE AUTHOR'S
INTERPRETATION, I'D LIKE TO ADD
THE GRAY FENCE ALSO REPRESENTS
TRANSPARENCY.
AND FROM AN ADVANCING EQUITY
PERSPECTIVE IF THERE'S A LACK OF
TRANSPARENCY HOW CAN ONE CHANGE
THE DISTRIBUTION OF WHAT THE BOX
REPRESENT TO RISE ABOVE THE
THRESHOLD AND BE EFFECTIVE ON
YOUR RESEARCH AND HAVE AN IMPACT
ON PUBLIC HEALTH.
SO, WHEN THE CONVERSATIONS
DIVERT TO EQUITY AND DISPARITIES
IN BIOMEDICAL RESEARCH, THE LENS
TEND TO FOCUS WHERE THERE IS
MORE PATIENT OR RESEARCH
PARTICIPANTS INTERACTION.
HOWEVER, IT IS IMPORTANT TO
UNDERSTAND THAT ADVANCING EQUITY
IS NOT CONSEQUENTIAL IN THE
CLINICAL AND RESEARCH REALM FOR
DISPARITIES BUT IN THE BASIC
PRECLINICAL AND TRANSLATIONAL
SPACE.
IT IS CRITICAL TO IDENTIFY
OPPORTUNITIES ALONG AND WITHIN
THAT THERAPEUTIC DEVELOPMENT
PIPELINE TO POSITION AND
IMPLEMENT DEEPER STRATEGIES.
SO WHAT SPACE ARE WE TALKING
ABOUT?
THIS IS ONE ILLUSTRATION OF THE
CONTINUUM OF THE BIOMEDICAL
RESEARCH ENVIRONMENT.
ALL OF
DPI BETWEEN THE BOOK ENDS OF
HEALTH CARE DELIVERY AND
CLINICAL RESEARCH AND TDB
OCCUPIES THE PRE CLINICAL SPACE
WITH VARIATION LINES ON OTHER
END.
AND WHAT COMES THROUGH
COLLABORATIVE PARTNERSHIPS.
TO THE EXTENT WE'RE PART OF THE
ECO SYSTEM WE NEED TO BE
COGNIZANT HOW WE ENDEAVOR TO
ENGAGE AND STRIVE TO BE MORE
EQUITABLE WITH OUR COLLABORATION
PARTNERS AND ON THE PROJECTS WE
UNDERTAKE.
SO, WHAT DO WE DO?
WE ENGAGE THE HEALTH OPERATED TO
WORK WITH US TO DEVELOP AN
APPROACH TO EQUITY INTO THE
PRECLINICAL THERAPEUTIC
DEVELOPMENT SPACE UTILIZING RARE
DISEASES AS AN APPROACH
CONSISTING OF FIVE STEPS I'VE
SUMMARIZED IN THE CHEVRON.
DEVELOP AN ACTION PLAN.
EXPLORE THROUGH AN ENVIRONMENT
STAND, APPLY CRITICAL EQUITY
LENS TO THE POINTS OF ENGAGEMENT
IN TDB AND IMPLEMENT THE
RECOMMENDATIONS.
IT'S IMPORTANT TO UNDERSTAND
THIS IS NOT A ONE AND DONE.
STEPS 3-5 ARE INTENDED TO BE I
APROCESS OF ENGAGING, REVIVING
AND LEARNING TO TRULY COMMIT TO
ADVANCING EQUITY.
THIS THE OUTPUT PRODUCES SEVERAL
FINDINGS.
THE REMAINDER OF THE
PRESENTATION IS THE HIGH LEVEL
ACTIONS OUTLINED IN ORANGE WE'RE
WORKING ON IN RESPONSE TO THE
RECOMMENDATIONS.
AND THEY ARE THE EVALUATION
PROCESS FOR A NEW PROJECT,
COLLABORATIONS, ENGAGE THE
COMMUNITY AND SHARE KNOWLEDGE.
AND FOR THE FIRST ACTION WHICH
IS TO APPLY AN EQUITY LENS IN
THE EVALUATION PROCESS FOR
COLLABORATION.
THE FINDING IS DISEASES
AFFECTING UNDER SERVED
POPULATIONS STRUGGLE TO FIND TO
ATTRACT RESOURCES OR
INSTITUTIONAL SUPPORT FOR
PRECLINICAL DEVELOPMENT.
THE RECOMMENDATION IS TO AUGMENT
THE EVALUATION OF THE PROCESS
AND RESEARCH EFFECTIVENESS
OCCURS ABOVE THE FENCE.
AND SOCIETAL IMPACT ON PUBLIC
HEALTH.
TRANSPARENCY IS IT CRITICAL FOR
EQUITABLE ALLOCATION.
WE NEED TO SEE WHAT IS BEHIND
THE FENCE TO BETTER UNDERSTAND
THE PLURALITY FOR THOSE UNDER
SERVED AND UNDER REPRESENTED IN
RESEARCH.
IT'S NOT ENOUGH TO LOOK AT RARE
DISORDERS BUT FOR OUR PROPOSAL
AND EVALUATION PROCESS TO LIVE
UP TO THE IDEAL OF ALL
POPULATIONS AFFECTED BY A
DISEASE AND THOSE WHO MAY OR MAY
NOT BENEFIT FROM THE PROPOSED
TREATMENT NEEDS TO BE
IDENTIFIED.
AND THE DEMOGRAPHIC INFORMATION
THAT ACCOMPANIES THE
INFORMATION.
IT MEANS TO STRIVE FOR MORE
TREATMENT , MORE QUICKLY
CONSIDER WHEN ALLOCATING
RESOURCES FOR COLLABORATIVE
OPPORTUNITIES THAT HOLDS PROMISE
PROPOSALS THAT ARE NOT QUITE IND
READY FOR COLLABORATIONS THAT DO
REQUIRE CATALYTIC ENGAGEMENT TO
FULFILL GAPS AND ADVANCE
TRANSLATION ARE CONSIDERED SO
THAT THEY TOO CAN RISE ABOVE THE
VISIBILITY OF RESEARCH
EFFECTIVENESS.
IT ALSO MEANS TO DIVERSIFY OUR
EFFORTS IN TREATMENT MODALITIES
SO THERE'S A BALANCE INCLUSIVE
OF THERAPIES AND EQUAL ONE OR
FEW AND REPURPOSE OR REPOSITION
MOLECULES.
ANOTHER IMPORTANT ASPECT THAT IS
BOTH A CHALLENGE AND AN
OPPORTUNITY IS NCATS IS DISEASE
AGNOSTIC.
APPROXIMATELY 10,000 RARE
DISEASES AND OTHERS WE DON'T
KNOW SUCH AS COMMON DISORDERS WE
DON'T KNOW ANYTHING ABOUT ANY
GIVEN DISEASE.
IN ADDITION TO THE INFORMATION
PROVIDED IN THE PROPOSAL AND
THROUGHOUT THE EVALUATION
PROCESS WHAT CAN WE GET A MORE
CONSISTENT AND COMPLETE PICTURE
OF THE DISEASE LANDSCAPE AND
EXPLICITLY EQUITY BEYOND THE
SCIENTIFIC DIMENSIONS.
TO ADDRESS THAT SPECIFICALLY WE
CONCEPTUALIZE AN APPROACH TO
DEVELOP A PRECLINICAL EQUITY
INDEX OR PEI TO COMPLEMENT THE
EVALUATION PROCESS.
THE PRAECHLT IS AN INTERACTIVE
ACCATION THAT INTRODUCES A NEW
CRITERIA IN A BINARY
QUESTIONNAIRE AND THE PEI
PRODUCES QUANTITATIVE DIMENSION
TO EVALUATE EQUITY PROFILES.
FROM THE SCREEN SHOTS THE END
USER ENTERS A DISEASE AND
RESPONDS TO THE QUESTIONNAIRE
DIVIDED AMONGST THE NAME WILL
CATEGORIES ON THE RIGHT.
THE TWO ARE NOT INTENDED TO BE A
SOLUTION TO SPECIFIC PROBLEMS OR
PRESENT DIRECT GUIDANCE FOR
ACTIONS TO BE TAKEN.
ANOTHER FEATURE OF THE TWO IS
THE READ OUTS.
THIS IS THE OUTPUT.
ESSENTIALLY THE QUESTIONNAIRE
THAT IS ANSWERED WITH POSITIVE
YES ANSWERS ARE DISPLAYED IN A
RADAR GRAPH.
YOU CAN SEE THE EXAMPLE OF TWO
DISEASES AND THE AREA THAT IS
LARGER THAT REPRESENTS MOST THE
QUESTIONS WERE ANSWERED AND THAT
CATEGORY IS BEING -- THANK YOU
SO MUCH.
THAT REPRESENTS THAT CATEGORY.
AS YOU CAN SEE HERE AS YOU HOVER
OVER THE POINTS YOU CAN GET AN
INDICATION OF THE NUMBER OF
QUESTIONS THAT WERE ANSWERED.
AND ON THE RIGHT YOU CAN ALSO
SEE THAT THIS IS ONE PARTICULAR
DISEASE THAT DIDN'T HAVE A WIDE
AREA THAT THE COVERED.
ON THE LEFT YOU CAN ALSO OVERLAY
THAT GRAPH AS PART OF THE
OUTPUT.
THIS CAN GIVE A VERY GOOD
REPRESENTATION OF WHAT IS
HAPPENING IN THE SPACE FOR A
RESEARCHER AND IF YOU'RE
THINKING ABOUT A PROPOSAL THAT
IS NOT IND READY, THIS CAN GIVE
AN INDICATION OF WHAT WE CAN DO
TO HELP THAT PARTICULAR DISEASE
IN THAT SPACE.
ANOTHER FEATURE OF THE TOOL
ALLOWS USERS TO INPUT DATA ON
COMMUNITY RESEARCH FUNDING THAT
ALLOWS THE USERS TO PROVIDE THE
RELATIONSHIP BETWEEN DIAGNOSIS
AND TARGET DRUG ABILITY IS
DISPLAYED HERE.
FROM THE PREVIOUS SLIDE YOU CAN
INPUT THAT FUNDING INFORMATION
FROM AN NIH REPORTER AND PLOT
THAT AGAINST THE DIAGNOSIS AND
DRUG ABILITY DIMENSIONS.
MOVING ON THE SECOND ACTION IS
TO ENGAGE THE COMMUNITY.
THE FINDING IS THAT LIMITED
ENGAGEMENT WITH PATIENTS AND
DISEASE COMMUNITIES CAN OBSCURE
THEIR PRIORITIES FROM RESEARCH
INSTITUTIONS.
THE RECOMMENDATION IS TO SUSTAIN
AND EXPAND COMMUNITY ENGAGEMENT
TO ENSURE RESEARCH EFFORTS ARE
INFORMED AND FOCUS ON PATIENT'S
TOP PRIORITIES AND UNMET NEEDS.
THOUGH NCATS IS NOT SOLELY
FOCUSSED ON RARE DISEASES WE
EMPHASIS RARE DISEASE RESEARCH
BECAUSE OF THE UNMEET NEED.
THAT MEANS THE CONSTITUENCY
COMPRISES SEVERAL GROUPS OF
PATIENTS.
OUR APPROACH.
IF YOU RECALL FROM THE FIRST
SLIDE, EQUITY RECOGNIZES EACH
PERSON HAS DIFFERENT
CIRCUMSTANCES AND ALLOCATES THE
EXACT RESOURCES AND
OPPORTUNITIES NEEDED TO REACH AN
EQUITY OUTCOME.
IF YOU GET THE CHANCE PLEASE
LOOK AT THE VIDEO ON THE RIGHT
FROM THE U.K.
IT SUMMARIZES QUITE A BIT OF
THAT.
AND TO THAT END WE ESTABLISHED A
SEMINAR SERIES OF COLLABORATION
PROCESS THAT IS INCLUSIVE OF
MORE THAN JUST THE RESEARCHER'S
VOICE BUT THE PATIENT AND
ADVOCATE AND PHYSICIAN AND OTHER
PARTNERS OF INTEREST.
IT'S MEANT TO BE CONVERSATIONAL
AND EVOLVE AND WE ADAPT IT TO
THE SPEAKER'S PREFERENCE AND
HEAR FROM THE COMMUNITY AND
THEIR PERSPECTIVES AND WHAT
MATTERS AND WHAT IT MEANS AS A
COMMUNITY.
LET'S DISCUSS THE ACTION TO
SHARE KNOWLEDGE.
SPECIFICALLY, EASIER ACCESS TO
RESOURCES AND PROVIDING NEW
KNOWLEDGE RESOURCES AS IT TIES
TO RESEARCH AND DEVELOPMENT.
I WOULD LIKE TO ACKNOWLEDGE THAT
NCATS HAS MANY AWESOME DATA
TOOLS AND RESOURCES ALIGNED TO
SPECIFIC ASPECTS OF THE
TRANSLATIONAL SCIENCE PIPELINE.
ONE FINDING IS THAT PATIENTS,
COMMUNITIES AND ADVOCATES
FREQUENTLY FIND THEMSELVES
NAVIGATING THEIR DISEASES ON
THEIR OWN HAVING TO BECOME THEIR
OWN RESEARCHER, ADVISER, DATA
EXPERTS, SAMPLE COLLECTOR AND
KNOWLEDGE BROKER.
ONE EXAMPLE IS TO EXPAND ONE
RECOMMENDATION INVESTMENTS IN
LOUISVILLING AND EASIER ACCESS
TO IT FOR RARE DISEASES.
TDB IS DEVELOPING A RARE SOURCE
A BIO INFORMATIC FORM AND IS A
CONTINUAL WORK IN PROGRESS.
IN THE INTEREST OF TIME I WANTED
TO FOCUS ON ONE PARTICULAR MODGE
YOU'LL -- MODULE AND BRINGING IT
TOGETHER WITH PHENOTYPES, GENES,
PROTEINS AN RELATED CHEMISTRIES.
THOUGH THROUGH THESE
CONNECTIONS, WE BETTER
UNDERSTAND COMMONALITIES AMONG
DISORDERS AS COMMON MECHANISMS
AND APPROACHES TO PLATFORM
THERAPY DEVELOPMENT.
THE FOCUS FOR TODAY IS THE
LITERATURE A.I.
THIS MODULE IS TRULY AN
OPPORTUNITY TO LEVEL THE PLAYING
FIELD.
WHEN IT COMES TO GETTING ACCESS
TO PUBLISHED PEER REVIEW
INFORMATION ON RARE DISEASES.
DISEASES CAN HAVE MANY ALIASES
AND ALL THAT ARE KNOWN ARE
LINKED AND CURATED IN THE
BACKGROUND.
IF A USER ENTERS ONE, THE
ALGORITHM YOU DON'T HAVE TO KNOW
EVERY ALIAS.
WE'RE WORKING TOWARDS THAT.
IT'S A WORK IN PROGRESS AND IT'S
NOT PERFECT BUT WE'RE ATTEMPTING
TO DO THAT.
WE RECENTLY ADDED OPTION TO
FILTER AND CONTINUING TO IMPROVE
THE USER EXPERIENCE.
YOU CAN SEE THE TOP 10 JOURNALS
AND NUMBER OF PUBLICATIONS AND
WHICH JOURNALS PUBLISH MORE
OFTEN FOR THE RARE DISEASE ARE
ALSO SHOWN AT THE TOP.
IF YOU RECALL IN THE DIAGNOSIS
AND DRUGABILITY GRAPH WE CAN
ALSO LOOK AT RESEARCH
EFFECTIVENESS THROUGH THE NUMBER
OF PUBLICATIONS.
THAT DATA CAN BE PULLED FROM
HERE.
THE SECOND FINDING IS TO SHARE
KNOWLEDGE.
RESEARCH PARTNERS AND ADVOCATES
ARE UNFAMILIAR WITH SPECIFICS OF
PRECLINICAL ACTIVITIES AND
REQUIREMENTS AFTER INITIAL DRUG
DISCOVERY WORK.
THE RECOMMENDATION IS TO DEVELOP
AND DISSEMINATE EDUCATIONAL
RESOURCES THAT INTRODUCE
PRECLINICAL DEVELOPMENT PHASE
AND DETAILED GENERIC
REQUIREMENTS OF THE SUCCESSFUL
CANDIDATE.
NOT WITHIN THE POWER OF TDB TO
UNDERSTAND PROCESSES AND BEST
PRACTICES OF SUCCESSFUL
THERAPEUTIC DEVELOPMENT.
THE EFFORTS AND RESOURCES OF
THESE COMMUNITIES CAN TRANSLATE
TO REAL PROGRESS.
SO WE DEVELOPED A KNOWLEDGE
GUIDE THAT FOCUSES IN ON THE
SPECIFIC SCIENTIFIC ACTIVITIES
OF PRECLINICAL DRUG DEVELOPMENT
FROM CANDIDATE SELECTION TO IND.
WE CONCEPTUALIZED AND ADDRESSED
THE VOID IN KNOWLEDGE AND ON THE
SPECIFIC DETAILS OF THE SPACE.
IT'S OFTEN REFERRED TO AS THE
VALLEY OF DEATH BUT WE WANTED TO
PROVIDE A LANDSCAPE WHILE
CHALLENGING TO TA VERSE OB
STAC
STACKAL -- OBSTACLES AND LOOK AT
SUCCESS AND FAILURES AND I'D
LIKE TO ACKNOWLEDGE MY
COLLEAGUES WHO WORKED ON THE
PROJECT AND ALSO EXTENDED
ACKNOWLEDGEMENTS TO THE FOLKS
WHO WORKED ONLY THE SOURCE.
THANK YOU VERY MUCH.
>> THANK YOU.
ARE THERE ANY QUESTIONS?
>> GREAT PRESENTATIONS THANK
YOU.
I'M REALLY INTRIGUED BY THE WORK
THAT YOU'VE DONE THE PROTOTYPE
WORK YOU'VE DONE LOOKING AT
DIAGNOSIS VERSUS DRUG VERSUS NIH
SPENDING.
CAN YOU TALK A LITTLE BIT MORE
ABOUT HOW YOU USE THAT TOOL TO
INFORM NEXT STEPS?
>> YES.
I REFER TO US BEING DISEASE
AGNOSTIC AND UNDERSTANDING WHERE
THE RESOURCES HAVE BEEN PLACED
IN THE PAST WILL BE IMPORTANT
FOR HOW WE WOULD PUT OUR
RESOURCES ESPECIALLY FOR THE
RESOURCES THAT MAY NOT BE IND
READY AND THINKING ABOUT WELL,
THIS IS PROMISING.
AND UNDERSTANDING FROM THE
DIAGNOSIS AND DRUGABILITY ASPECT
WHETHER THE TARGETS ARE READY
FOR THE INVESTMENT OR TAKE A
STEP BACK AND HOW MUCH FUNDING
IS OUT THERE WITH ACADEMIC OR
OTHER INSTITUTIONS THE
GOVERNMENT HAS ALREADY PROVIDED.
WE CERTAINLY DON'T WANT TO
DUPLICATE EFFORTS.
ALL THAT IS IMPORTANT TO KNOW
AND USING A TOOL LIKE THAT IS
HELPFUL.
>> QUICKLY FOLLOWING UP ON THAT
ONE.
THE SIZE OF THE CIRCLES IN THAT
GRAPH WITH THE AMOUNT OF
FUNDING.
I THINK WHAT I'M HEARING YOU SAY
IS LOOKING FOR PATTERNS ON THE
X, Y AXIS WHERE THEY FALL AND IF
YOU SEE LITTLE CIRCLES WHERE
THERE SHOULD BE BIG CIRCLES IS A
RIPE AREA FOR INVESTMENT.
>> SO LET ME TAKE A STEP BACK.
THE PURPLE COMES TO US AND THEY
IT'S NOT A COMPARATIVE TOOL FROM
THAT PERSPECTIVE.
WHEN WE LOOK AT THE INDIVIDUAL
DISEASE -- AGAIN, THIS TOOL CAN
BE USED BY WHOEVER.
ANYONE CAN APPLY THIS TO THEIR
AREA BUT WHAT WE WANT TO
UNDERSTAND IS WE HAVE A CERTAIN
AMOUNT OF RESOURCES.
WE WANT TO UNDERSTAND THE
DISEASE AREA AND LANDSCAPE AND
YOU'RE LOOKING AT THE SIZE OF
THE FUNDING AND IS THE TARGET
READY FOR THAT INVESTMENT OR DO
WE NEED TO TAKE A STEP BACK?
YES, THAT COULD BE INDICATIVE OF
WHERE FUNDING COULD BE APPLIED.
THAT'S HOW WE INTEND TO USE IT.
COMPARING THE PURPLE IS NOT
SAYING OKAY GIVE ME DISEASE A
AND Y AND DECIDE THAT.
THAT'S NOT OUR INTENT.
>> I'D LIKE TO ADD PERHAPS MAYBE
THERE WILL COME A DAY TO HAVE
THE ABILITY AND RESOURCES TO BE
ABLE TO TAKE A LOOK AT SOME
GRAPHS LIKE THAT THAT PERHAPS
AREN'T BEING STUDIED BUT WE
MIGHT BE ABLE ADVANCE FOR
REASONS THAT ARE BEING UNDER
STUDIED FOR PARTICULAR REASONS
OR THERE'S AN EQUITY ISSUE WE
CAN PERHAPS INCREASE THE
KNOWLEDGE AROUND.
IF WE DO GET THIS WOULD SHINE A
LIGHT.
>> YOU CAN ADAPT IT FOR THEIR
OWN PURPOSES AND THERE'S AN
ASSESSMENT OF WHAT WAS PROPOSED.
>> A COMMENT.
I ENJOYED LEARNING ABOUT THE
TOOL.
ONE EXAMPLE SHE GAVE ME AS SHE'S
BEEN EDUCATING ME ON THE PROGRAM
SINCE I BECAME S.D.
IF SOMEONE COMES WITH A
CANDIDATE THERAPEUTIC FOR A
GIVEN TARGET THAT HELPS THEM
ZOOM OUT AND ASK WHAT OTHER
PATIENTS AND THINK ABOUT ALL PEOPLE AND ALL DISEASES AND
HELPED ME CONTEMPLATE AS DR. HAUGABOOK MADE CLEAR TO LOOK AT ALL THE INTRAMURAL PROGRAM.
IT'S NOT MEANT TO BE
COMPREHENSIVE.
IT CAN'T BE, UNFORTUNATELY.
WHILE WE LIKE TO SAY WE'RE
DISEASE AGNOSTIC WE'RE NOT
DISEASE ATHEISTS.
WE HAVE A WIDE VARIETY OF
DISEASES WE ARE IN FACT
ADDRESSING.
WE HAVE WHAT STAFF PUSHES AND
WHAT MY ECLECTIC TASTE FINDS
INTERESTING.
THIS IS BASICALLY TO REMIND
PEOPLE THAT RIGHT NOW THOUGH
BUDGET PEOPLE DOESN'T SHOW UP
AND WE HAVE A BUDGET IN PROCESS
AND COMING OFF AN FY24 A 0%
INCREASE.
[RECORDING IN PROGRESS]
>> WE'RE DOING WHAT WE CAN AND
THIS IS WHAT THE FY24 CTSA
PROGRAM LOOKS LIKE AS OF NOW.
I THINK EVERYTHING THAT'S
SUPPOSED TO GO OUT THE DOOR WILL
GO OUT THE DOOR OR WILL BY
FRIDAY.
UNDER OUR NEW UM1 PROGRAM WE HAD
11 CTSAs AWARDED IN FY24, I'M
HAPPY TO SAY.
DARTMOUTH HAD BEEN A PREVIOUS
ONE YEARS AGO BUT SUCCESSFULLY
CAME BACK IN AND DOWN IN
HOUSTON, BAYLOR IS OUR NEWEST
CTSA.
WITHIN OUR STAFF WHERE HE ADDED
TWO PEOPLE WITHIN OUR EDUCATION
AND TRAINING SESSION AND ANTHONY
AND KARINA HAS JOINED THE
CLINICAL AFFAIRS BRANCH AS AN
ADVISER AND HEATHER BAKER HAS
MOVED UP TO NOW FROM A HEALTH
POLICY ANALYST TO SENIOR PROGRAM
COORDINATOR IN THE OFFICE OF THE
DIRECTOR AND PROVIDING MUCH
NEEDED COORDINATION ACROSS THE
PROGRAM.
I'D LIKE TO START WITH
CONGRATULATIONS.
WE HAVE A NUMBER OF SIGNIFICANT
PLAYERS WITHIN THE CTSA.
SO JULIE AT VANDERBILT WAS
ELECTED VICE PRESIDENT OF THE
AMERICAN SOCIETY FOR CLINICAL
INVESTIGATION AND WILL
SUBSEQUENTLY SERVE AS PRESIDENT
ELECT AND THEN PRESIDENT.
SO, SHE'S BEEN OBLIGATED THE
NEXT SEVERAL YEARS.
SHE'S CURRENTLY THE P.I. FOR KL1
AND 2 AT VANDERBILT.
RHONDA IS AT CONVENIENCE AND
RECIPIENT OF THE CYSTIC FIBROSIS
CLINICIANS REWARD AND VESNA THE
P.I. AT MAYO AND FROM THE
RECIPIENT OF AWARDS AND NICOLE
IS THE EXECUTIVE DIRECTOR AT
NORTHWESTERN AND WAS AWARDED THE
AMERICAN SOCIETY FOR BIO
CHEMISTRY AND EMERGING
LEADERSHIP AWARD AND AT THIS
YEAR'S ACTS MEETING THOSE WHO
WENT HELD IN LAS VEGAS.
I'M TRULY HOPING IF IT'S EVER
HELD IN LAS VEGAS AGAIN I'LL BE
RETIRED BY THEN.
TERRENCE TSOU WON THE
OUTSTANDING TRAINING PREDOCTORAL
TRAINING AWARD AND A TRAINEE AT
JOHNS HOPKINS.
MOVING TO A NATIONAL LEVEL, ERIC
TOPOL PROBABLY ONE OF THE MAJOR
CROWN TOOLS PART OF THE SCRIPPS
RESEARCH WAS NAMED BY TIME AS
ONE OF THE MOST INFLUENTIAL
PEOPLE IN HEALTH AND WON THE
FRIEND OF THE NATIONAL OF
LIBRARY MEDICINES DISTINGUISHED
HEALTH COMMUNICATIONS AWARD.
AND THIS ONE IS INTERESTING.
DAVID HUANG AT OREGON SEE HIM
WINNING THE NATIONAL MEDAL OF
TECHNOLOGY AND INNOVATION AND HE
WON IT LATE LAST YEAR AND WAS
TOO LATE TO INCLUDE AT LAST
YEAR'S COUNCIL MEETING.
HE IS BEING RECOGNIZED FOR HIS
INITIAL WORK BACK IN '91 ON
OPTICAL COHERENCE TOMOGRAPHY
WHICH IS BASICALLY AN AMAZING
TECHNOLOGY THAT IF UTILIZED
MILLIONS OF TIMES A YEAR IN EYE
EXAMS.
HIS INVOLVEMENT WITH THE OREGON
CTSA IS PILOT AWARD AND THIS WAS
DEVELOPED FOR EYE EXAMINATIONS.
HE UTILIZED PILOT AWARDS TO LOOK
AT CARDIOVASCULAR AND CARDIOLOGY
AND DERMATOLOGY.
IT'S A SIGNIFICANT TECHNOLOGY IN
TERMS OF IMAGING POTENTIAL.
FINALLY, ELIZABETH OFILLI AT
MOREHOUSE COMPLETELY
INDEPENDENT.
WE WERE NOT CONTACTED UNTIL
ELIZABETH CALLED ME AND SAID
I'LL BE UP IN WASHINGTON AT THE
WHITE HOUSE.
CAN I STOP BY AND SEE YOU?
THAT'S HOW WE LEARNED.
THE OSTP AT THE WHITE HOUSE
ORGANIZED AN EVENT THAT WAS
TALKING ABOUT INNOVATIVE
APPROACHES TO MAKE INCLUSIVE
TRIALS MORE INCLUSIVE AND
ACCESSIBLE AND HIGHLIGHTED THE
HELP OF 360 A MOBILE AND
WEB-BASED REGISTRY WITH
CONGRUENT COACHING.
THIS WAS INITIALLY SUPPORTED AS
A SUPPLEMENT TO THE GEORGIA CTSA
AND FUNDED AS AN SBIR THROUGH
NCATS.
IT'S VERY GRATIFYING WHEN
OUTSIDE GROUPS LIKE THIS REALLY
RECOGNIZE THE UTILITY AND VALUE
OF WHAT WE'RE DOING THERE'S
PROBABLY MORE BUT THERE'S MORE
WE'VE BEEN INFORMED OF.
WE SUNK OUR TOE IN THE WATER IN
TERMS OF AN R PROGRAM AND WE
HAVE FOR FY24 THE AWARDEES.
THIS IS LIMITED COMPETITION TO
CTSK AND DIVERSITY SCHOLARS AND
WHAT YOU CAN SEE IS THERE'S A
WIDE RANGE OF ACTIVITIES FROM
SOME MODULATION OF T CELL
DIFFERENTIATION AND ANTI-TUMOR
IMMUNITY ALL THE WAY THROUGH THE
ENROLLMENT IN HEALTH SPENDING
AND UTILIZATIUTILIZATION.
A WIDE FACTOR OF ACTIVITIES
VALUABLE FOR THE PROGRAM.
OUR CCIAs WHICH ARE MECHANISM
FOR ALLOWING MULTIPLE CTSAs TO
COME TOGETHER TO WORK ON A JOINT
PROJECT THAT WOULD BE VERY
DIFFICULT FOR ANY INDIVIDUAL
CTSA TO BE CARED ABOUT.
WE HAD TWO.
ONE IS BYTES TO BEDSIDE FOR
CLINICAL TRANSLATIONAL SUPPORT.
I'LL IS PAUL IS THIS INFORMATICS
OR DATA SCIENCE.
WE HAD AN INTERESTING
CONVERSATION.
THE IDEA HERE IS AND THEY'VE
DUNK THE KOOL-AID.
AND DEVELOP, DEMONSTRATE AND
DISSEMINATE AND EVALUATE A
CENTRALIZED AND HIGHLY EFFICIENT
DATA SHARING INFRASTRUCTURE TO
SUPPORT TRANSLATIONAL CLINICAL
DECISION SUPPORT ANALYSIS.
SIX INSTITUTIONS ARE INVOLVED
WHICH I THINK IS A REAL CRITICAL
NE
NEED.
A SING AT INSTITUTION HAS
LIMITED AABILITY.
THIS IS IMPORTANT TO SHOW THE
UTILITY ACROSS MULTIPLE
INSTITUTIONS.
THE SECOND CCIA AWARD IS
FOCUSSED ON ANOTHER CRITICAL
ASPECT WE PUT EFFORT INTO IN
RECENT YEARS.
THIS IS IMPROVING EFFICIENCY,
QUALITY AND EQUITY, RANDOMIZED
REMOTE VERSUS CLINICAL IN TRIAL
METHODS.
CLEARLY COVID'S LED TO A
FOCUSSED EFFORT AROUND REMOTE
AND THERE'S A LOT OF ACTIVITIES
GOING TO ON IN THIS.
WHILE I DON'T THINK REMOTE WILL
GO WE AND CONVERT TO REMOTE
VERSUS IN PERSON I THINK IT'S
IMPORTANT WE UNDERSTAND THE
LIMITS AND THE POSSIBILITIES
WITH EACH OF THE DIFFERENT
MODALITIES GOING FORWARD SO IN
THE FUTURE WE CAN DESIGN TRIALS
OPTIMALLY TO TAKE THE FACTORS
INTO ACCOUNT.
WANTED TO HIGHLIGHT ONE PRIOR
CCIA.
I LIKE TO POINT OUT PAST ONES
BECAUSE WE SOMETIMES TELL YOU
ABOUT THE WONDERFUL THINGS WE'RE
FUNDING BUT WE DON'T NECESSARILY
TELL YOU WHAT THEY'VE DONE.
SO THIS IS AN INTERESTING ONE
BECAUSE IT ACTUALLY DOVETAILS
QUITE NICELY WITH JONI'S SLIDE
EARLIER ON THE VENTURE SPACE FOR
NEWBORN SCREENING.
THIS IS PRECISION MEDICINE AND
DIAGNOSIS OF GENETIC DISORDERS
IN NEONATES.
THIS IS AN AWARD BACK WE
INITIATED IN 2018.
WHILE IT'S NOT THE FIRST, IT IS
TO DATE THE LARGEST STUDY, 400
STUDENTS.
INFANTS THAT WERE BIRTHED THAT
END UP IN THE NICU AND COMPARE
WHOLE GENOME SECOND-CHANCE FOR
THE PANEL ANALYSIS FOR THE
POSSIBLE DISORDERS.
ALMOST HALF, 49% OF THOSE 400
WERE IDENTIFIED WITH A SPECIFIC
GENETIC DISORDER.
ABOUT 1 IN 5 RESULTED IN
CHANGING THE CARE THEY WERE
RECEIVING.
THE WHOLE GENOME SEQUENCING
WHILE IT MAY HAVE BEEN A DAY OR
TWO LATER THAN PANEL ANALYSIS,
IT CLEARLY WAS SUPERIOR SO
THERE'S VERY GOOD RCT DATA THAT
SHOWS THIS IS IN FACT SUPERIOR.
MANY PRIOR STUDIES LOOKED AT THE
COST OF CARE WITH WHOLE GENOME
INCLUDE FOR THE INITIAL STAY.
THEY DID A FOLLOW-UP ANALYSIS IN
THE FIRST YEAR OF LIFE AND FOUND
THE OVER ALL SAVINGS FOR THE
FIRST ENTIRE YEARLY OF THE
INFANT'S LIFE WAS OVER $150,000
IN TERMS OF CARE BECAUSE OF THE
WHOLE GENOME SEQUENCING.
AND WHEN I SPOKE TO STEVEN
KINGSMORE ABOUT THE PROGRAM I
SAID, WHAT ABOUT THE OTHER 51%
WHERE YOU DIDN'T IDENTIFY
GENOMIC ABNORMALITIES IS IT
POSSIBLE WE'LL IDENTIFY BENEFITS
AND HE SAID IT'S INTERESTING YOU
SAY THAT BECAUSE WE SECURED
ADDITIONAL FUNDING SO WE'RE
GOING TO TRACK THESE KIDS OVER
THE NEXT FIVE YEARS TO SEE IF
THEY COME DOWN.
IF WE RECOGNIZE SOMETHING THAT
THEY HAVE BECAUSE IT'S BEEN
SOMETHING THAT'S REPORTED IN THE
LITERATURE.
THE ARTICLE ON THE PUBLICATION
TON THE RIGHT-HAND SIDE AN
AUTOMATED 13.5 HOUR SYSTEM FOR
SCALABLE DIAGNOSIS CLEARLY I
DONT THINK THE EXPECTATION IS TO
HAVE A CHILD'S GENOME SEQUENCE
PRODUCED WHILE A CHILD IN THE
NICU AND HAVE THE CLINICIAN
TRYING TO FIGURE OUT WHAT IT ALL
MEANS.
THEY'VE CREATED AN AUTOMATED
SYSTEM WHERE THE SEQUENCING IS
DONE AND YOU CAN SEE THEY REFER
TO IT AS A GENOME TREATMENT AND
EVALUATING NEARLY 10,000 DRUG
DEVICES, DIETARY INTERVENTIONS
AHAVE OVER 1500 INTERVENTIONS SO
IT'S AUTOMATED.
ONCE IT'S DONE THE ANALYSIS CAN
TELL YOU IF YOU FALL INTO THE
SET, THIS IS WHAT YOU NEED TO
DO.
THIS IS NOT JUST BRINGING
SEQUENCING IN BUT HOW WE CAN USE
IT AND USE IT EFFECTIVELY ACROSS
A BROAD HEALTH CARE SYSTEM OR
CURRENT BALKANIZED SYSTEM.
WE HAVE CONTINUED TO DELIVER
OVER AND OVER AGAIN WITH N3C.
ONE Cs IS COVID.
WE'RE CONVERTING THAT TO
CLINICAL.
N3C IS STILL LIVING.
WE CONCEPTUALIZED THIS AND SENT
NOTICES ASKING FOR THE
INDIVIDUAL INSTITUTIONS
SUPPLYING DATA IF THEY WOULD SO
CHOOSE THEY COULD RE-ENLIST FOR
ANOTHER FIVE YEARS.
I CAN SAY MOST HAVE DONE THAT.
N3C COVID CONTINUES TO REMAIN
OPEN.
AND IN OUR TRANSLATION TO
CLINICAL WE HAVE THREE PILOTS
GOING ON.
ONE IS THE DATA SCIENCE ENCLAVE
WAS SOMETHING ODSS, THE NIH DATA
SCIENCE GROUP, CAME TO US AND
WAS LOOKING FOR AN EDUCATIONAL
RESOURCE.
ONE OF THE PROJECTS WITHIN N3C
HIT AND UTILIZED WITH THE
CREATION OF SYNTHETIC DATA.
THE ADVANTAGE IS THOUGH IT
EXACTLY REPLICATES WHAT IS
WITHIN AN E.H.R. YOU DO NOT NEED
TO GO TO AN IRB IN TERMS OF
USING IT SO IT'S AN EDUCATIONAL
TOOL AND A LARGE DATA SET THAT
BECOMES AVAILABLE FOR PEOPLE TO
LEARN HOW TO BEGIN WORKING ON
THE LARGE DATA SETS.
WE'RE NOT THE ONLY ONES
INTERESTED IN THIS.
I SHOULD ADD THE ONC, OFFICE OF
THE NATIONAL COORDINATOR HAS
BEEN VERY MUCH INTERESTED IN
SYNTHETIC DATA SETS BECAUSE
VENDORS WHO WOULD LIKE TO
OPERATE WITH E.H.R. DATA SHOW
THEY HAVE SOFTWARE AND NEED
SOMETHING TO WORK WITH AND THE
LAST THING YOU WANT TO DO IS
GIVE PERSONAL DATA.
SYNTHETIC DATA SET ARE USEFUL.
AND EXTEND THE ABILITIES OF
SYNTHETIC DATA.
THE OTHER A RENAL AND CANCER
ENCLAVE.
THERE'S A WHOLE ALPHABET SOUP OF
DIFFERENT GROUPS THAT HAVE BEEN
INTERESTED IN THIS NOT JUST
WITHIN NIH BUT OBVIOUSLY WITH
RENAL WE HAVE NIDDK AND WITH
CANCER WE HAVE NCI BUT OTHER
GROUPS INCLUDING THE FDA AND THE
ASSISTANT SECRETARY FOR PLANNING
AND EVALUATION AT HHS HAVE BEEN
VERY MUCH INTERESTED IN THESE
TYPES OF APPLICATIONS AND HAVE
PONIED UP FUNDING TO SUPPORT
THESE EFFORTS.
SO THESE PILOTS ARE REALLY A
CRITICAL EVALUATION TO
UNDERSTAND THE REQUIREMENTS FOR
THE INFRASTRUCTURE NEEDED TO
SUPPORT THIS TYPE OF WORK AS
WELL AS RESOURCES INSTITUTIONS
NEED TO BE ABLE TO TAKE
ADVANTAGE MUCH IT.
I THINK IT'S DEMONSTRATING THE
ABILITY OF THE LINKAGE OF
MULTIPLE DATA SETS TO PROVIDE
VALUABLE AND USEFUL INFORMATION
AND I THINK WE'VE GOTTEN
INQUIRIES ABOUT DIFFERENT
ENCLAVES.
ONE ADVANTAGE IS BECAUSE OF THE
HARMONIZATION TECHNOLOGY N3C
UTILIZES TO CONVERT THE
INDIVIDUAL E.H.R.s TO A SINGLE
STANDARD, WHAT IT MEANS IS THAT
IF AN INSTITUTION WERE TO BE IN
THE RENAL ENCLAVE, THEIR ENTIRE
E.H.R. DATA IS HARMONIZED AND
WHAT'S EXTRACTED IS JUST A
SPECIFIC RENAL PATIENT
INFORMATION THAT IS NECESSARY
BUT IF THEY CHOOSE AT A LATER
POINT TO ENTER THE ENCLAVE THEIR
DATA IS HARMONIZED SO THREES A
SCALABILITY HERE THAT MAKES IT
EASIER TO ADD MORE AND MORE
ENCLAVES AS WE GO.
THERE'S STILL AN INFRASTRUCTURE
AND COMPUTING COSTS ARE
SOMETHING THAT HAVE TO BE DEALT
WITH.
WITH THE ENCLAVES THEY'RE
TEACHING US AND WILL DEMONSTRATE
THE POWER OF WHAT BIG DATA CAN
ACCOMPLISH.
THESE ARE ALL CENTRALIZED
DATABASES AND WE HAVE A
FEDERATED EFFORT.
THE ENACT SYSTEM.
THEY'VE BEEN BRINGING THEMSELVES
ONLINE BUT THERE'S NOW MORE THAN
50 CTSAs UTILIZING OVER 140
MILLION PATIENT E.H.R.s.
THE IDEA WHILE THERE'S CERTAINLY
THE CAPABILITY FOR RESEARCH,
THIS WAS ALSO WITH THE INTENTION
TO MAKE THIS MORE PRACTICAL FOR
INDIVIDUAL CLINICIANS.
SOMEONE DOES NOT HAVE TO BE A
Ph.D. DATA SCIENTIST OR Ph.D.
INFORMATIC SPECIALIST TO TAKE
ADVANTAGE.
THESE THINGS SHOULD BE MUCH MORE
USER AVAILABLE IN TERMS OF
DECISION MAKING CAPABILITY.
WANTED TO HIGHLIGHT SOMETHING
THAT WAS STARTED PRE COVID AND
SIDE TRACKED A LITTLE BIT
BECAUSE OF COVID BUT HAS DONE
NICELY.
THE OPIOID CRISIS LEADING TO THE
NIH HEAL ACTIVITIES.
WE PRODUCED THE MAIN MANAGEMENT
EFFECTIVENESS RESEARCH NEXT OR
PAIN ERN.
THE IDEA IS THERE'S MANY AREAS
OF THE BIOMEDICAL SPACE THAT DO
NOT HAVE AN OBVIOUS HOME AT HNI
AND PAIN IS ONE OF THOSE.
ALMOST EVERY I.C. HAS PAIN.
BUT WE DON'T HAVE AN NATIONAL
INSTITUTE OF PAIN IN ANY WAY.
SO THE ABILITY FOR PAIN TO BE
EFFECTIVELY THE SPECTRUM OF PAIN
IS VARIABLE.
IF YOU'RE A WELL RESOURCED I.C.
LIKE SAY NCI YOU MAY BE ABLE TO
HANDLE THIS BUT OTHER SMALLER
I.C.s HAVE DIFFICULTIES.
YOU CAN SEE THE PARTNERS
INVOLVED.
THE FOUR BLUE ONES YOU SEE ALL
FULLY ENROLLED THOSE WERE
STARTED IN 2019.
THE ONE BEGAN IN 2020 AND GOT
MORE SIDE TRACKED BECAUSE OF
COVID IS NEARING COMPLETION.
THE SICKLE CELL WAS NOT AWARDED
UNTIL FY23 AND ANTICIPATE FIRST
PATIENT IN PROBABLY NEXT MONTH.
THE THREE LOWER ONES ARE WHAT WE
REFER TO AS LIFE TOUCH.
THEY DIDN'T INVOLVE THE FULL
PAIN NETWORK BUT WE DID PROVIDE
RESOURCES TO HELP THEM ACHIEVE
IT.
YOU CAN SEE EVEN WITH COVID THE
COMPLETION RATE HAS BEEN
ABSOLUTELY PHENOMENAL IN THIS
REGARD AND THERE'S MUCH
SATISFACTION ACROSS NIH FOR THE
ABILITY OF DIFFERENT INSTITUTES
TO BE ABLE TO PARTICIPATE IN
THIS EFFORT SUCH THAT WE'VE BEEN
ASKED TO BEGIN CONSIDERATIONS OF
RE-COMPETITION FOR THIS IN THE
NEXT COUPLE YEARS.
WE HAVE QUITE A BIT OF
RECOGNITION BEYOND NCATS.
WE DO HAVE A NUMBER OF
ADMINISTRATIVE SUPPLEMENTS THAT
COME FROM ACROSS NIH.
YOU CAN SEE NIDA PROVIDED SOME
FUNDING FOR US AND THEN A NUMBER
OF TRANS-NIH ACTIVITIES LARGELY
COMING FROM THE OFFICE OF
SCIENCE POLICY OR THE OF THE OF
THE DIRECTOR AT NIH.
THE TWO INCLUDE PART OF A DOWN
SYNDROME PROGRAM TRANS NIH AND
THEY'VE BEEN PLEASED WITH OUR
INVOLVEMENT IN THAT OVER THE
LAST SEVERAL YEARS.
I'M GOING TO RUN THROUGH
HIGHLIGHTS.
THESE ARE THINGS PEOPLE HAVE
BROUGHT TO MY ATTENTION RANDOMLY
PIQUED MY INTEREST.
SO THIS IS AN INTERESTING STORY
BECAUSE IF YOU LOOK AT THE
ATHEROSCLEROSIS ARTICLE ON THE
RIGHT PUBLISHED IN 2021, THE TWO
MAJOR OMEGA 3 FATTY ACIDS
REFERRED TO AS FISH OIL DIFFER
IN THEIR BEHAVIOR.
THE INTERESTING THING IS THIS
WAS NOT FUNDED BY NIH BUT THE
USDA.
I WOULDN'T HAVE THOUGHT TRADE BE
INTERESTED BUT THEY WERE.
THE SUBSEQUENT RESULTS OF THE
TRIALS LED TO A SERIES AND THE
2024 ARTICLE ON THE LEFT THEY'VE
ENGAGED CTSA S AND THIS IS SAY
COLLABORATION AND THE
INTERESTING FEATURE WITH THE
ANALYSIS IS WHILE EPA AND DHA
DIFFER IN TERMS OF BEHAVIOR,
WHICH MEANS IF YOU'RE LOOKING AT
CARDIOVASCULAR STUDIES THAT USED
FISH OIL WHETHER YOU USE PURE
EPA OR DHA OR A MIXTURE OF JUST
REGULAR FISH OIL YOU'LL GET
DIFFERENT RESULTS.
WHAT THEY'VE SHOWN RECENTLY IS
THAT MEN AND WOMEN DIFFER IN HOW
THEY RESPOND TO THIS.
THIS PROBABLY UNDER LIES THE
OBSERVED VARIABILITY IN RESPOND
TO EPA AND DHA IN WOMEN CAN
EXPLAIN CONFLICTING RESULTS IN
CARDIOVASCULAR DISEASE FROM
OTHER TRIALS.
THIS IS SORTING OUT WHY THERE'S
A DIFFERENCE IN REPORTING ON THE
EFFECTS OF EPA AND DHA.
AND IT'S MEN AND WOMEN DIFFERING
IN HOW THEY RESPOND AND IN THE
CASE OF MEN THEY TEND TO HAVE I
AGREATER PRODUCTION PRORESULTING
POLARIZATION AND WOMEN HAVE A
GREATER REDUCTION IN
PRO-INFLAMMATORY SIGNALLING
WHICH MEANS THERE MAY BE VERY
DIFFERENT RISK FACTORS GOING ON
IN THESE TWO.
THIS SHOULD BE TAKEN INTO
CONSIDERATION IN TERMS OF FUTURE
TRIALS.
WE HAVE A VERY INTERESTING GROUP
AT THE UNIVERSITY OF NEW MEXICO.
MATTHEW CAMPIN IS THE P.I. AND
HAS BEEN A LEADER IN THE MICRO
NANO PLASTICS ISSUE WE'RE
HEARING SO MUCH ABOUT.
THIS IS FROM A RECENT ARTICLE
THEY HAVE IN TERMS OF
QUANTIFICATION.
THEY PUT A LOT OF EFFORT INTO
DEVELOPING THE ASSAYS THAT ARE
ABLE TO MEASURE THE MICRO
PLASTICS IN A VARIETY OF TISSUE
AND LOOK AT THE LIGHT BLUE
COLUMN WITH THE BRAIN AND SHOULD
SEND RED FLAGS IMMEDIATELY AND
GET PEOPLE TO NOTICE.
THIS IS SOMETHING THAT I THINK
WILL GET MORE ATTENTION AND
NILES -- NICE TO SEE THE CTSA
DRAWING ATTENTION TO IT.
AT WASH-U THEY ARE DEVELOPED A
DIAGNOSTIC TEST BASED ON GENOME
SEQUENCING FOR MYELOID PLASTIC
SYNDROME AND LEUKEMIA CHOMEMOSI
THEY BECAME THE FIRST WHOLE
GENOME TESTING TO BE APPROVED BY
CMS.
IT'S SIGNIFICANT -- CHROMO SEQ
AND WE'RE SEEING THIS ACTUALLY
MANIFEST TO PATIENT CARE.
THIS IS AN INTERESTING STUDY OUT
OF DAVIS.
SO I CAN SAY THAT BACK WHEN I
WAS IN MEDICAL SCHOOL, GRANTED
WAS A LONG TIME AGO, I'LL ADMIT.
I WAS ONCE A CAESAREAN ALWAYS A
CAESAREAN AND THERE'S A TRIAL OF
LABOR ACT CAESAREAN.
RATHER THAN HAVING A WOMAN DO A
CAESAREAN AGAIN IT'S GIVING THE
OPTION OF DOING A TRIAL OF
LABOR.
THERE IS SOME CONCERN BUT THERE
HAD BEEN A CALCULATOR AND IN
2022 THE NICHD RELEASED
CALCULATOR WITH RACE AS A
VARIABLE AND THE UC DAVIS CTSA
WENT ON TO VALIDATE THE
CALCULATOR WITH THEIR OWN DATA
AND THEY FOUND THE RISK IT
SEEMED TO WORK AS WELL IN TERMS
OF PREDICTING RISK AND THE
AVERAGE PREDICTED SUCCESS OF
WHAT IS THE TRIAL OF LABOR AFTER
CAESAREAN IMPROVED FOR BLACK AND
HISPANIC WOMEN.
I THINK THEY ARE SATISFIED AND
UTILIZING AND COULD ADOPT THIS
MOVING IT FORWARD.
SERGIO TAUGHT ME THE DIFFERENCE
BETWEEN TRUST AND
TRUSTWORTHINESS.
THIS IS FROM THE UNIVERSITY OF
KENTUCKY CTSA AND IDENTIFYING
CHURCH LEADERS AND UNDERSTANDING
THEIR PERSPECTIVE IN TERMS OF
COLORECTAL CANCER SCREENING
PARTICULARLY FOR AFRICAN
AMERICAN PEOPLE FROM KENTUCKY
AND UTILIZING THE ADVANTAGES OF
THE CHURCH AS A TRUSTED CENTER
FOR COMMUNITY SUPPORT AND
EMPOWERMENT.
CLEARLY COLORECTAL CANCER
SCREENING IS INTENSE AND
INVOLVED AND YOU OBVIOUSLY NEED
QUITE A BIT OF SUPPORT AND
COACHING IN ORDER TO GET THROUGH
AND I THINK THE KENTUCKY CTSA AS
USED A CHURCH COMMUNITY AS A
MEANS FOR ACTUALLY IMPROVING
COLORECTAL SCREENING RATES.
I WANTED TO TALK ABOUT OUR MOST
VALUABLE PLAYERS, THE YOUNGER
CREW.
TO HIGHLIGHT ONE PARTICULAR KL2
SCHOLAR, CHIDIOGO AT CINCINNATI
CHILDREN'S HOSPITAL NAMED EMORY
UNIVERSITY'S NOTABLE 40 UNDER 40
ALUMNI AND SHE IS A KL2 SCHOLAR
CURRENTLY.
AND ALWAYS LIKE TO HIGHLIGHT
WHERE PEOPLE GO AFTERWARDS
WHAT'S IT MEAN TO HAVE A KL2
SCHOLAR.
THIS WAS A SCHOLAR FROM 2018 TO
2020 AND NOW THE ASSOCIATE
PROFESSOR OF ANESTHESIOLOGY
HUMAN IMPACT LAB AT WEILL
CORNELL AND HAS BEEN AWARDED THE
2023 NATIONAL ACADEMIES HEALTH
AND MEDICINE LONGEVITIES PRIZE
AND DIGITAL SOLUTIONS TO REDUCE
MATERNAL MORBIDITY AND MORTALITY
IN WOMEN AND THIS IS A MATTER OF
MEETING PEOPLE WHERE THEY ARE.
IN TERM OF T TRAINEES, CARLY IS
THE FIRST AUTHOR ON AN NIH
FUNDED RADX PROJECT.
THE INTERESTING RESULT OF THE
STUDY IS IT WAS LOOKING AT VIRAL
LOAD ACROSS INDIVIDUALS WITH
COVID.
AND FOUND THERE WAS A VIRAL LOAD
INCREASED AMONG BMI AMONG MALES
BUT IT REMAINS STABLE AMONG
FEMALES WHICH PLAY UNDER LIE
DIFFERENCES IN THE EFFECTS OF
VIRAL INFECTION DIFFERENCES
BETWEEN MEN AND WOMEN.
IT'S AN INTERESTING ANALYSIS.
SO IN TERMS OF A FORMER TL1
SCHOLAR.
ALISON IS A DOUBLE DIPPER
BECAUSE SHE WAS A T SCHOLAR AT
UCSF AND BECAME A CASE SCHOLAR
AT NORTHWESTERN AND NOW THE
ASSOCIATE DEAN OF RESEARCH AND
PROFESSOR OF CHILD, FAMILY AND
POPULATION HEALTH NURSING AT THE
UNIVERSITY OF WASHINGTON SCHOOL
OF NURSING.
SHE'S DONE QUITE A BIT IN THE
HIV FIELD.
YES, THE PEOPLE WE HIGHLIGHT WE
DO SUPPORT GO ON TO BIGGER AND
BETTER THINGS.
FOR OUR FY24 DIVERSION TI AND
RE-ENTRY SUPPLEMENT SCHOLARS WE
HAVE SIX.
TO HIGHLIGHT TWO, SUELEN IS
FOCUSSED ON PARKINSON'S DISEASE
AND DEEP BRAIN STIMULATION.
AND DA SHAUNDA LOOKS AT
MATERN
MATERNITY.
THIS PUBLICATION THAT CAME OUT
WHICH IS REDUCING DIAGNOSTIC
DELAYS IN ACUTE HEPATIC DISEASES
USING HEALTH RECORD DATA AND
MACHINE LEARNING THE SECOND
AUTHOR IS IS LOOKING AT THIS
ACUTE HEPATIC PORPHYRIA AND IT
CAN TAKE VERY LONG BECAUSE
SYMPTOMS ARE RELATIVELY NON
SPECIFIC.
SO THE ALGORITHM THEY DEVELOPED
WHICH IS INTRIGUING BECAUSE
RATHER THAN JUST LOOKING AT
POSITIVE AND NEGATIVE TEST
RESULTS THE IDEA IS LET'S LOOK
AT THE TESTS ORDERED TO GET AN
IDEA OF WHAT THE DIFFERENT
CLINICIANS WERE THINKING ABOUT
AND SEE IF WE CAN PUT IT ALL
TOGETHER AND COME UP WITH A
DIAGNOSIS EARLIER.
THE IDENTIFIED 71% OF THE CASES
A YEAR EARLIER THAN WHAT WAS
DONE CLINICALLY.
SHE WAS A SUMMER STUDENT WITH A
MEDICAL STUDENT AND DID THIS AS
A SUMMER PROJECT.
SHE JUST GRADUATED THIS PAST
MAY.
I'LL HAVE TO ALERT THE P.I. AT
DARTMOUTH AND SHE'S UP THERE NOW
BECAUSE IF YOU LOOK AT HER
LINKEDIN PROFILE THESE ALONG THE
LINES OF WHAT WE'RE TRYING TO DO
HERE.
THE GETS IT.
SHE AND TWO OTHER INDIVIDUALS
FOUNDED WHAT IS NOW CALLED ZEBRA
MD DOT ORG AND THEY HAVE THE TOP
THREE AFTER HEPATIC PORPHYRIA
AND THE DISEASE INDEX IS
INCREDIBLY LONG.
THE WEBSITE IS UP AND RUNNING
AND IT'S REALLY EXPLORING THE
POWER OF E.H.R.s TO IDENTIFY
UNRECOGNIZED AND UNDIAGNOSED
DISEASES AT THE THIS POINT.
AND TRAINING THE NEXT GENERATION
OF CUTTING EDGE PHYSICIAN
SCIENTISTS.
I HIGHLIGHT UNIVERSITY OF
PENNSYLVANIA HAS AN
UNDERGRADUATE TRANSLATIONAL
IMMERSION PROGRAM.
IN A SURVEY OF 22 PEOPLE WHO
HAVE GONE THROUGH THIS FROM THE
SURVEY THERE WERE 23 INDIVIDUALS
WHO GRADUATE PROGRAMS AND HALF
M.D.s.
THESE ARE TWO EXAMPLES.
SARA RAMIREZ AND JACKSON MALONE
ON THE TWO PAPERS LOOKING AT
GENE AND ENVIRONMENT S IN
MEDIATION INVOLVING ADVERSE
CHILDHOOD EVENTS, MOOD AND
ANXIETY DISORDERS AND A
PHENOTYPE SAMPLE AND SUBJECT USE
DISORDERS.
SARA IS AN A GRADUATE STUDENT IN
NEUROSCIENCE AND JACKSON JUST
STARTED HIS SENIOR YEAR.
AND THIS IS A NEW YORK
UNIVERSITY LANGONE CTSA.
PRECEDENCES IN HISTORICALLY
REDLINED DISTRICTS IN NEW YORK
CITY AREA HAVE MORE INDOOR
PARTICULATE AIR POLLUTION
POTENTIALLY REDUCIBLE BY
PORTABLE AIR CLEANERS.
WHILE WE FIND A LOT OF ISSUES WE
FIND IN TERMS OF REDLINE
DISTRICTS THIS IS ACTUALLY
IDENTIFYING WHAT IS A RELATIVELY
STRAIGHTFORWARD CORRECTIBLE
ASPECT OF PARTICULATE SOLUTIONS
AND IT'S ASSOCIATED WITH A WIDE
VARIETY CARDIOVASCULAR AND
PULMONARY PROBLEMS LATER IN LIFE
AND IS READILY ADDRESSABLE BY
CLEANING THE AIR.
THE TWO WON AND THEY WERE HIGH
SCHOOL STUDENTS AND THEY'RE
GIVING A PRESENTATION HERE OF
THEIR WORK.
THIS WAS A PILOT AWARD THROUGH
THE NYU.
AND LASTLY I'LL FINISH UP SO
KIDS SPEND TIME PLAYING VIDEO
GAMES AND MANY OF THEM ARE
PROBABLY FLYING UNMANNED DRONES
FOR THE MILITARY AT THIS POINT
BUT I THINK WHAT WE'RE MOVING
INTO IS A NEW ERA.
SO ANU HAS BEEN PLAYING AROUND
WITH DEEP LEARNING MODELS SINCE
SHE WAS 14 YEARS OLD AND
CURRENTLY AN UNDERGRADUATE AT
GEORGIA INSTITUTE TECHNOLOGY AND
THE FIRST AUTHOR OF AN
UNIVERSITY OF ARKANSAS
PUBLICATION THAT DETECTS
PARKINSON'S DISEASE INVOICE
RECORDINGS WHICH I THINK IS
INCREDIBLY INTRIGUING BECAUSE
IT'S VERY ATYPICAL FROM WHAT WE
WOULD EXPECT.
A LOT OF PEOPLE I SEE AND MY
COLLEAGUES THINK OF A.I. OF
DOING SOMETHING I DO NOW FASTER
OR WITH FEWER PEOPLE IF YOU'RE
TALKING ABOUT ANALYZING X-RAYS
OR SOMETHING LIKE THAT BUT THIS
IS DOING SOMETHING ENTIRELY NEW.
SOMETHING ENTIRELY DIFFERENT.
I DON'T THINK ANYBODY WOULD HAVE
THOUGHT YOU WOULD DO THIS.
I DON'T KNOW IF PEOPLE WHO
SPECIALIZE IN PARKINSON'S WOULD
AGREE OR WOULD THINK THIS IS AT
ALL POSSIBLE.
INTERESTINGLY THERE'S A SMALL
COMPANY IN CANADA THAT HAS
PUBLISHED WORK IN BEING ABLE TO
DIAGNOSIS HIGH BLOOD PRESSURE
AND TYPE II DIABETES BY VOICE
RECORDINGS AS WELL.
WE MAY BE ON THE CUSP OF
APPROACHING HOW IT'S REIMAGINING
HOW WE DELIVER HEALTH CARE.
NOW JUST GETTING SOMEBODY TO
TALK TO YOU MAY BE ENOUGH TO
PROVIDE VALUABLE DIAGNOSTIC
INPUT INTO WHAT IS GOING ON WITH
THEM AND SO I THINK THESE
OPPORTUNITIES FOR HIGH SCHOOL
KIDS ARE REALLY -- IT REMINDS ME
OF THIS STEVE JOBS COMMENT HE
SAID WHEN HE FIRST RELEASED THE
MAC BOOK AND GAVE IT TO OLDER
ADULTS THEY ASKED WHAT DOES IT
DO BUT WHEN HE GAVE IT TO
CHILDREN, CHILDREN ASKED WHAT
CAN I DO WITH IT.
I THINK THAT'S WHAT WE'RE
LOOKING FOR.
LASTLY TO HIGHLIGHT THE FALL
MEETING WILL BE HERE IN EVERYONE
SAYS NORTH BETHESDA.
I PREFER TO SAY SOUTH BROCKVILLE
NOVEMBER 14th THROUGH THE 15th.
THAT'S IT.
>> THANK YOU, MIKE.
THANK YOU FOR THE TOUR DE FORCE
OF THE EARLY CAREER INDIVIDUALS.
I'D LIKE TO OPEN IT UP FOR
QUESTIONS FROM COUNCIL.
ANY QUESTIONS?
GO AHEAD.
>> THIS MAY BE TOO PROVOCATIVE
FOR 3:00 IN THE AFTERNOON.
FIRST IT WAS A PHENOMENAL
PRESENTATION.
THE WORK IS INCREDIBLE, THANK
YOU.
JUST A THOUGHT IN THINKING ABOUT
THIS OPPORTUNITIES OF MACHINE
LEARNING PRESENTATION AND FOR
CONTEXT I'M DEEPLY INVESTED IN
SHORTENING THE DIAGNOSTIC
ODYSSEY ESPECIALLY IN RARE
DISEASE AND LED WORK IN RARE
DISEASE MYSELF.
ONE OF THE THINGS THAT OCCURS TO
ME AND I LOVED YOUR FRAMING THAT
LAST QUESTION BEYOND WHAT CAN I
DO BUT WHAT CAN I DO WITH IT,
ONE OF THE THINGS I WORRY ABOUT
AND I THINK ABOUT IS HOW DO WE
ENSURE WE PROTECT PEOPLE FROM
THOSE WHO WILL USE THE
TECHNOLOGY NOT FOR GOOD BUT FOR
HARM.
AND SO IN THE PATIENT COMMUNITY
AND IN THE ECO SYSTEMS WE HAVE
PRACTICES THAT ARE VERY HARMFUL
AS PRODUCTS ARE MOVING FROM
REGULATORY TO ACCESS PACES AND
HEALTH INSURERS NOW CAN
ANTICIPATE THERE ARE PRODUCTS
CAN LASER COMMUNITIES OUT OF
THEIR PLANS SO THEY DON'T HAVE
TO PAY FOR AND REIMBURSE FOR
THOSE PRODUCTS BY CUTTING THE
PRODUCT OUT OF THEIR PLAN OR THE
DISEASE IN A PARTICIPATORY WAY.
AND WHAT I WORRY IT MAY BE A
HEALTH CARE SYSTEM THAT'S PART
OF THESE TECHNOLOGIES, HOW LONG
WILL IT BE BEFORE PEOPLE START
TO USE THE TECHNOLOGY NOT JUST
TO SHORTEN THE DIAGNOSTIC
ODYSSEY WHICH IS INCREDIBLY
IMPORTANT AND SUPPORT SHORTENING
ODDITIES AND OPTIMIZING OUTCOMES
AND GETTING TREATMENTS TO PEOPLE
FACIL
FASTER AND INTERVENTIONS AND NOT
JUST APPROVED TREATMENTS BUT
THIS TECHNOLOGY COULD BE USE TO
IDENTIFY PEOPLE BEFORE THEY HAVE
A DIAGNOSIS AND ELIMINATE THEM
FROM A HEALTH CARE ENVIRONMENT
OR MILLION.
I WONDER WHERE THE FUND BEING OF
THAT SCIENCE POLICY AND THE
RESPONSIBILITY TO FUND THE
POLICIES THAT PROTECT PATIENTS
FROM THAT ALSO RUNS ALONGSIDE
THIS.
WHILE WE NEED IT MAKE SURE WE
USE THE TECHNOLOGY TO SOLVE FOR
THE DIAGNOSTIC ODYSSEYS WE HAVE
TO MAKE SURE WE PROTECT PEOPLE
WHO DON'T EVEN KNOW THE
TECHNOLOGY IS BEING ADVANCED AND
DEVELOPED.
THIS IS HAPPENING AND IT WILL BE
HAPPENING AND I ASK THE QUESTION
OF IS THAT AN NCATS
RESPONSIBILITY FOR SOMEBODY
ELSE'S RESPONSIBILITY BUT NEEDS
TO BE SOMEBODY'S RESPONSIBILITY.
>> YOU THINK IN MANY WAYS WHAT
YOU'RE REALLY ASKING IS SIMILAR
TO WHAT WAS INITIALLY FEARED
WITH THE BRINGING ON BOARDS AND
THE ABILITY TO DO GENOME
SEQUENCES AND SOMEHOW I THINK WE
HAD THE NAIVE APPROACH THAT
MAYBE A SMALL FRACTION OF THE
POPULATION WILL HAVE GENETIC
DISORDERS AND EXCLUDED FROM
HEALTH INSURANCE BECAUSE IT
WOULD BE CALLED A QUOTE,
PREEXISTING CONDITION.
CONGRESS HAS SAID YOU CAN'T DO
THAT BUT SUBSEQUENTLY WHAT WE
LEARNED IN TERMS OF GENOME
SEQUENCING IS AND WE'RE NOT AND
THERE'S GENETIC MUTATIONS THAT
ALL HAVE A SUBTLE IMPACT ON
INFLUENCE THE RISK FACTORS WHICH
IS WHY THERE'S A VARIETY OF
CONDITIONS.
SO I THINK FROM THE GENETIC
STANDPOINT AND THAT WAS SOLVED
BY CONGRESS SIMPLY SAYING
ELIMINATE THE PREEXISTING
CONDITIONS.
I THINK WHAT YOU'RE GETTING AT
IS WITH A.I. YOU HAVE THE
POTENTIAL TO DEVELOP ALGORITHMS
WHETHER THE MEDICAL COMMUNITY
WANTS THEM OR USING THEM TO
POTENTIALLY CAUSE PROBLEMS.
I THINK THAT'S A REALISTIC
POSSIBILITY.
IN MANY WAYS ULTIMATELY IT COMES
DOWN TO WHEN I TALK ABOUT
REIMAGINING ABOUT HEALTH CARE
I'M TALKING ABOUT HOW WE
RE-ENVISION OUR HEALTH CARE
SYSTEM AND WE AT LEAST THE
ACADEMIC COMMUNITY NEEDS TO BE
PROVIDING THE EVIDENCE TO
SUPPORT WAYS TO BE OPERATING.
NOW IN THE END THAT'S A POLICY
DECISION AND IT'S WELL BEYOND
THE ACADEMIC OR MEDICAL
COMMUNITY TO MAKE THIS CALL BUT
I THINK IT BEHOOVES US TO
PROVIDE RIGOROUS EVIDENCE TO
SUPPORT THE MIGRATION TO WHAT
WOULD BE A MORE EQUITABLE HEALTH
CARE SYSTEM THAT WOULD BE
BENEFICIAL TO EVERYONE.
>> YEAH, AND I WOULD JUST SAY I
AGREE WITH YOU BUT THE
ELIMINATION OF PREEXISTING
CONDITIONS AND GENETIC NON
DISCRIMINATION WERE LAWS AND
PROTECTIONS PUT IN PLACE BY
CONGRESS BECAUSE THE COMMUNITY
BROUGHT THAT AS PROTECTIONS THAT
NEEDED TO BE IN PLACE INCLUDING
THOSE AT NIH AND FRANCIS COLLINS
WAS A PART OF THE LEADERSHIP AND
THE PROTECTIONS COULD BE
REVERSED.
AND SO I'LL PLUG THAT THE
COMMUNITY IS DEVELOPING THE
SCIENCE WHEN EXTENDED
PROTECTIONS MIGHT BE NECESSARY.
>> I THINK IT'S A GREAT COMMENT
AND IT REMINDS ME OF THE
DISCUSSIONS THAT WE HAD WITH FDA
AND CMS.
IT'S NOT THE SAME THING BUT IT'S
REMINISCENT OF THE TREATMENTS
BEING DEVELOPED FOR COMMUNITIES
OR WITH RARE DISEASES AND THE
CLINICAL TRIALS NOT NECESSARILY
REFLECTING THE AGE GROUPS THAT
ARE IN NEED OF SAID THERAPIES.
SO WE'RE NOT QUITE ON THE RIGHT
TRACK HERE FOR DECISIONS TO BE
MADE ABOUT ACCESS TO THOSE
THERAPIES BECAUSE WE HAVE TESTED
THEM.
AND THINK ABOUT THE FORWARD
LOOKING POLICIES THAT MIGHT ALSO
BE NEEDED.
AND I THINK THAT THESE ARE
QUESTIONS WE'RE STARTING TO ASK
AND THOSE SORTS OF MODALITIES
BUT ALSO NEED TO DO IT IN THE
DIGITAL HEALTH SPACE AND FOR US
THE COMMENT IS JUST A REMINDER
THAT THIS IS SOMETHING WE NEED
TO THINK ABOUT AS WE START TO
SUPPORT MORE OF THESE ACTIVES TO
AN INDIVIDUAL SPACE.
>> I THINK ONE OF THE THINGS I
THINK IF WE TEST AN INTERVENTION
THE CRITERIA FOR THAT IS THE
RIGOROUS DESIGN AND EXECUTION OF
THAT TRIAL THAT'S GOING TO GET
IT PUBLISHED IN THE JOURNAL LIKE
NEW ENGLAND JOURNAL OF MEDICINE
AND YET WHAT I THINK WE DON'T
ALWAYS THINK ABOUT IS THAT OTHER
ENTITIES LIKE INSURERS, CMS, IF
YOU'RE TALKING ABOUT SOMETHING
IN THE OPIOID REALM MAY BE LAW
ENFORCEMENT OR THE JUSTICE
COMMUNITY, THOSE ENTITIES HAVE
DIFFERENT STANDARDS OF EVIDENCE
WE NEED TO UNDERSTAND SO THAT WE
CAN DESIGN TRIALS SO THAT IT
WILL NOT ONLY GET THE TRIAL
PUBLISHED AND RECOGNIZED BY THE
ACADEMIC AND MEDICAL COMMUNITY
BUT RECOGNIZED AS EVIDENCE FOR
THOSE OTHER COMMUNITIES BECAUSE
MORE OFTEN NAN NOT, WHAT ENDS UP
HAPPENING IS WE PUBLISH A STUDY.
THE MEDICAL COMMUNITY SAYS YES
AND THEN CMS SAYS, YEAH, BUT YOU
DIDN'T DO THIS AND THIS.
THE INSURERS SAY YOU WERE
WORRIED ABOUT THIS BUT YOU
DIDN'T DO THAT.
IT'S INTERESTING IN TERMS OF
VACCINES, FOR EXAMPLE, YOU KNOW
GETTING PAST THE FDA INCREDIBLY
HIGH BAR BUT TO VACCINE
MANUFACTURERS THERE'S ACTUALLY A
HIGHER BAR THE ADVISORY COUNCIL
AT IMMUNIZATIONSS AND THEIR
RECOMMENDATIONS THAT DETERMINES
WHO WILL BE REIMBURSED.
THE FDA PLAY SAY THIS IS A
PROVED BUT IF ACIP DOESN'T
RECOMMEND THAT FOR THAT
PARTICULAR POPULATION IT WON'T
BE REIMBURSED OR USED.
THE RECOMMENDATIONS ARE
INSUFFICIENT TO GET IT PAST ACIP
AND IT'S INCUMBENT TO RECOGNIZE
THEY'RE ADDRESSING MORE THAN THE
MEDICAL COMMUNITY AND JOURNALS.
WE NEED TO REACH OUT AND
UNDERSTAND ALL THESE DIFFERENT
GROUPS.
WE TALK ABOUT ENGAGING WITH
PATIENT GROUPS AND NEED TO THINK
ABOUT ENGAGING WITH INSURERS,
CMS, ALL THESE OTHER GROUPS TO
UNDERSTAND THEIR REQUIREMENTS
AND THEIR NEEDS.
>> ANY OTHER QUESTION FOR MIKE?
WE'RE A LITTLE BIT AHEAD OF
SCHEDULE BUT ALSO BEHIND
SCHEDULE.
THAT DOESN'T MAKE SENSE.
BECAUSE WE HAD A 10 MINUTE BREAK
IF YOU'D LIKE TO PROCEED OR PUSH
FORWARD.
IF YOU WANT A FIVE-MINUTE BREAK
WE CAN DO THAT NOW OR PUSH
FORWARD.
WE'LL TAKE A FIVE-MINUTE BREAK
AND THEN COME BACK.
AND WE'LL GO TO THE NEXT
PRESENTATION.
THANK YOU.
>> CHRISTINE CLOVIS WILL KICK US
OFF AND INTRODUCE KARLIE.
>> THANK YOU.
I AM CHRISTINE CLOVIS.
I THINK EVERYBODY HERE KNOWS ME
BUT MAYBE FOLKS ONLINE DON'T.
I'LL TEE SOME STUFF UP FOR
KARLIE.
SHE'LL GIVE US A NICE CLOSE UP
REPORT ON THE GENOME PROGRAM.
A PROGRAM ONGOING ABOUT 10 YEARS
NOW.
O AND THEN IF THERE'S TIME I'LL
COME BACK AND GIVE A TEASER WHAT
YOU MIGHT HERE ABOUT NEXT TIME
NEXT YEAR.
FIRST, WE HAVE FOUR PROGRAM
OFFICERS.
THERE'S INCREDIBLY PRODUCTIVE
THANKS TO THE OFFICE OF
COORDINATOR AND PROGRAM ANALYST.
WE'RE FORTUNATE FOR A COUPLE
YEARS WE'VE SHERAT WORKING WITH
US ON THE BIOMEDICAL TRANS DATA
PROGRAM WITH THE OFFICE OF DATA
STRATEGY AS A DATA SCHOLAR.
HE'S BEEN WORKING WITH US THE
LAST TWO YEARS.
HIS TENURE IS ABOUT TO END AND
AARON BERNSTEIN HAS BEEN A DATA
SCHOLAR FOR 10 MONTHS THE PAST
YEAR ALSO AS A DATA SCHOLAR.
AND WE BREAK IT DOWN FROM BIG
ANNOUNCEMENTS AND SHOW GENERAL
AREAS.
FIRST WE HAVE EXPANDING THE
TARGET LANDSCAPE FOCUSSED ONLY
EARLY DISCOVERY TYPE THINGS.
IT WILL BE THE FOCUS OF WHAT YOU
HEAR ABOUT TODAY.
AND THEN DRUG DEVELOPMENT
PARTNERSHIPS THAT WOULD PICK UP
A BALL FROM THERE ONCE WE HIT
DEVELOPMENT AND IDENTIFY EARLY
PHASE TO CLINICAL TRIALS AND IT
TAKES SKILL SETS AND THE PEOPLE
WHO DO TALKS ARE NOT THE SAME
WHO DO THE CHEMISTRY.
IT TAKES A TEAM.
WE I HAVE TO DEVELOP PROGRAMS
THAT WILL SUPPORT AND ENCOURAGE
AND INCENTIVIZE PEOPLE TO
ACTUALLY WORK TOGETHER TO DO
THAT DEVELOPMENT.
AND WE HAVE TECHNOLOGY
DEVELOPMENT AND ASSETS PROGRAM
AND OUR TECHNOLOGY FOR
TRANSLATIONAL SCIENCE THAT CAN
SPAN THE ENTIRE PIPELINE AND SO
AS I SAID THE THEME WILL BE
EXPANDING THE TARGET LANDSCAPE
IN WHAT YOU'LL HEAR IN CONCEPT
CLEARANCE.
SO WE TAKE A TWO-PRONGED
APPROACH WHEN WE THINK ABOUT
EXPANDING THE TARGET LANDSCAPE.
ONE IS TO EXPLORE UNDER STUDIED
DRUGGABLE PROTEINS.
A LOT OF THE WORK STARTED UNDER
THE DRUGGABLE GENOME PROGRAM THE
IDEA IS YOU FOCUS ON PROTEINS
THAT HAVE A BINDING SITE INTO
WHICH A SMALL MOLECULE, CHEMICAL
COULD MODULATE RECEPTORS,
ENZYMES, ION CHANNELS.
THE BOTTOM LINE IS IT MAKES IT
ACCESSIBLE AND THE SECOND IS
APPROACH IS DRUGGING THE
UNDRUGGABLE.
THE THINGS THAT DON'T HAVE
BINDING SITE FOR US TO ACTUALLY
CREATE A SMALL MOLECULE FOR.
THEY COULD BE NON-PROTEIN
TARGETS AND WE HAVE TOUGH
TARGETS.
SO THESE ARE THE TWO PRONGS FOR
EXPANDING THE LANDSCAPE AND
YOU'LL HEAR FROM KARLIE IN A
COUPLE MINUTES AND WHEN WE GET
TO THE CONCEPT CLEARANCE, THE
CONCEPT WE'RE CLEARING IS TO
ADDRESS THE SECOND PRONG
APPROACH.
SO, FOCUSSING NOW ON THE FIRST
PRONG ELIMINATING THE DRUGGABLE
GENOME.
THINK OF IT AS THE LOW HANGING
FRUIT WHEN IT COMES TO DRUG
DEVELOPMENT.
WE HAVE THE CONVENIENT BINDING
SITE AND LOOKING AT UNDER
STUDIED DRUGGABLE PROTEINS.
WHEN WE SAY UNDER STUDIED WE
MEAN THINGS FOR WHICH THERE ARE
VERY FEW PUBLICATION OR RO1 OR
SUBSTANTIAL SUPPORT FROM THE
NIH.
THE IEG PROGRAM WAS TO STIMULATE
RESEARCH ON UNDER STUDIED
LOW-HANGING FRUIT.
THIS IS AN NIH COMMON FUND
PROGRAM.
COMMON FUND PROGRAMS TYPICALLY
HAVE A 10 YEAR LIFE SPAN.
THEY CAN ONLY LAST FOR 10 YEARS.
THEY HAVE FIVE CRITERIA THEY
REALLY AIM TO MEET TO FIT THEIR
MODEL WHICH IS THE PROGRAM HAS
TO BE CATALYTIC, GOAL DRIVEN,
SYNERGISTIC WITH THE OTHER
INSTITUTES AND SCIENCE THAT'S BE
DONE ACROSS THE NIH
TRANSFORMATIVE AND NOVEL.
I THINK OF THE PROGRAM AS BEING
VERY TRANSFORMATIVE.
IT WAS DESIGNED TO SUPPORT THE
EXPLORATION OF THE UNKNOWN.
IT'S SUPPORTING A GENERATION OF
PRELIMINARY DATA FOR SUBSEQUENT
AWARDS.
THIS IS WHERE I FEEL IT CAN BE
TRANSFORMATIVE AND GET US OUT OF
THE MODE OF ME TOO DRUGS AND
WORK ON THINGS THAT MOVE BEYOND
THAT.
AGAIN IT'S RELATIVELY LOW
HANGING FRUIT.
THE IG COORDINATOR IS
RESPONSIBLE FOR COORDINATION OF
A LARGE TRANS-NIH TEAM OF
PROGRAM STAFF, SENIOR STAFF AND
OVERSIGHT OVER THE PROGRAM.
ONE THING I WANT TO MENTION THE
PROGRAM FOR SMALL AWARDS THE
SUCCESS WE SAW WITH THE MODEST
INVESTMENTS $100,000 IT'S A YEAR
AND WE SAID LET'S A VERSION AT
NCATS.
NOW WE HAVE THE ONE WE DO AT
NCATS FOCUSSED ON RARE DISEASES.
DRUGGABLE PROTEINS THAT ARE
ASSOCIATED WITHRARE DISEASES.
WITH RARE DISEASES.
SO WITH THAT I'LL HAND IT OVER
TO KARLIE.
>> OKAY.
I JUST WANT TO MENTION THAT THE
DRUGGABLE GENOME EXPRESSES
PROTEINS POTENTIALLY ABLE TO
BIND DRUG LIKE COMPOUNDS.
WHILE THE EXISTING NUMBER OF
PROTEINS IS ESTIMATED TO BE
BETWEEN 3,000 AND 4500, WE ONLY
HAVE EXISTED PHARMACOPEIA FOR A
FEW HUNDRED OF THE TARGETS.
THERE'S A LOT OF BIOLOGY WE
HAVEN'T EXPLOITED YET.
SO THE GOAL OF THE PROGRAM WAS
TO IMPROVE UNDERSTANDING OF
PROPERTIES AND FUNCTIONS OF
PROTEINS NOT WELL STUDIED WITHIN
PROTEIN FAMILIES.
QUE LOOKED AT THE ION CHANNELS
AND KINASES AND G PROTEINS AND
THIS IS A COMMON FUND AND THIS
HAS BROAD APPLICABILITY.
AS THE PROTEINS ARE VERY UNDER
STUDIED THEY DON'T HAVE A CLEAR
FIT WITHIN AN I.C. MISSION.
WE RECOGNIZE THROUGH DISCOVERY
OF THE ROLE WE CAN GET A HANDLE
ON THE FUNCTION AND WHERE THEY
FIT WITHIN NIH AND DESPITE SO
MANY DRUG PROTEIN FAMILIES
THERE'S PROTEIN FAMILIES AND
WANTED TO TEST THE IDEA IT WOULD
STIMULATE RESEARCH AND OVER ALL
IT'S BEEN A SIGNIFICANT SUCCESS.
I SHOW THE SLIDE TO GIVE AN IDEA
OF EXACTLY HOW WIDE SPANNING THE
PROGRAM WAS.
WE HAVE INDIVIDUALS FROM ALL
OVER THE DIFFERENT INSTITUTES
AND CENTERS INVOLVED, OUR
WORKING GROUP SPANNED DIFFERENT
AREAS.
WE ALSO A MULTI INSTITUTE GROUP
SO THERE WAS INTEREST FROM A LOT
OF GROUP.
AND THIS IS THE TIME LINE AN
BUDGET.
WE HAD A PILOT PHASE OF 25.5 TO
ADAPT SCALABLE IDG FAMILIES AND
PLATFORM AND MOVED TO THE
IMPLEMENTATION PHASE IN 2018
WITH THE $71 MILLION TO IDENTIFY
PHENOTYPES OF THESE UNDER
STUDIED PROTEIN FAMILIES.
THE ONES FROM PREVIOUS SLIDE AND
ALSO PROVIDE TOOLS AROUND THE
PROTEIN FAMILIES TO THE
SCIENTIFIC COMMUNITY.
WE'RE ALSO WORKING TO CREATE AN
ENRICHED KNOWLEDGE BASE WITH
SUBSEQUENT DATABASE BEHIND THIS
I'LL GO OVER AND MAKE YOU AWARE
OF THE MAIN GOALS IN THE
PROGRAM.
AND WHAT I THINK THIS TELLS US
IS THAT NCATS IS LEADING THE WAY
IN IDENTIFYING THE DARK AND LOST
PROTEINS AND PUTTING THEM IN THE
CONTEXT OF HUMAN DISEASE.
SO THE PROGRAM DEPENDED ON THE
CONSORTIUM STRUCTURE FOR
SUCCESS.
EACH COMPONENT REQUIRED THE
EFFORTS OF THE OTHERS IN ORDER
TO EFFECTIVELY GENERATE THE
RESOURCES, PROVIDER RESOURCES
PUBLICALLY IN AN ORGANIZED
MANNER AND ALSO ENSURE THE
PUBLIC WAS AWARE OF THE
RESOURCES BY OUR OUTREACH.
SO THERE WERE THREE MAIN TYPES.
THE FIRST WAS DATA AND RESOURCE
GENERATION CENTERS.
ONE FOR EACH OF THE DIFFERENT
PROTEIN FAMILIES AND THESE ARE
THE WET LABS.
THE ONCE MOSTLY IN CHARGE OF
GENERATING THE RESOURCES SUCH AS
THE MOUSE MODELS AND THINGS
ALONG THOSE LINES.
WE HAD THE KNOWLEDGE MANAGEMENT
CENTER.
THE BIO INFORMATICS WING AND
THEY FOCUSSED ON THE INTERPHASE
AND RESOURCE DISSEMINATION
CENTER WHICH WITH US CRITICAL IN
TERMS OF THE ADMINISTRATION AND
OUTREACH PART OF THE PROGRAM.
OUTSIDE THE CENTERS WE ALSO HAD
A NUMBER OF OTHER AWARDS THAT
WENT OUT.
WE HAD CUTTING EDGE INFORMATICS
TOOLS AWARD TO HELP THE
SCIENTIFIC COMMUNITY BETTER
ANALYZE AND VISUALIZE DATA AND
THE PROGRAM I'LL GO MORE IN
DEPTH ON IN A LITTLE BIT.
THE PURPOSE WAS TO HELP
ELUCIDATE THE DARK PROTEINS IN
THE CONTEXT OF HUMAN DISEASE.
FINALLY, WE HAD SOMETHING WE DID
ADJACENT TO THE IDG PROGRAM.
THIS WAS THE SPIR FUNDING BUT
FOR A SMALL BUSINESS FUNDING
OPPORTUNITY BASED ON THE
PROTEINS THAT WERE HIGHLIGHTED
IN THE IDG PROGRAM.
ESSENTIALLY COMMERCIALIZING SOME
ASSAYS AND RESOURCES AROUND
THOSE UNDER STUDIED PROTEINS.
JUST TO GIVE AN IDEA OF THE
IMPACT WE'VE HAD ON THE FIELD.
ON THE LEFT IS AN INCREASED IN
PUBLICATION AROUND THE IDG UNDER
STUDIED PROTEINS.
THE RED LINE IS THE
IMPLEMENTATION PHASE WHEN THEY
STARTED.
I WANT TO EMPHASIZE THIS
ANALYSIS DOES NOT INCLUDE
PUBLICATIONS FROM THE AWARDEES
TO THE IDG PROGRAM.
WE DID THAT BECAUSE WE DON'T
WANT TO SKEW THE DATA.
THIS IS ONLY GOING TO THE
PROGRAM.
YOU CAN SEE THE PUBLICATIONS
AROUND THESE UNDER STUDIED
PROTEINS DID AND YOU CAN SEE A
NICE STEADY PUBLICATION RATE
THROUGHOUT THE SPAN OF THE
PROGRAM AND THE CRITATIONS
CONTINUE TO INCREASE EVERY YEAR.
WE'RE CONSIDERING INTEREST
AROUND THE PROGRAM.
I WANT TO GIVE YOU AN IDEA OF
THIS INTERFACE.
THIS IS SOMETHING DEVELOPED BY
THE DEVELOPING GROUP AND PULLS
DATA NOT JUST FROM IDG BUT OTHER
COMMON FUND PROGRAMS AND KIMBALL
AND PUB MED AND THERE'S I THINK
80 RESOURCES AT THIS POINT BUT
FEED INTO THE INTERFACE TO
SEARCH THE ENTIRE HUMAN GENOME
IN HERE NOT JUST THE UNDER
STUDIED PROTEINS.
SO YOU CAN GO HERE AND SEARCH
AND IT WILL GIVE ALL THE
INFORMATION THAT HAS BEEN
CLEANED FROM THE 80 PLUS
RESOURCES.
WE ALSO HAVE THE CAPACITY FOR
SEARCHS ASSOCIATED WITH
DISEASES.
THIS IS UNIQUE TO THESE ARE WHAT
WE CONSIDER DARK VIRTUALLY
NOTHING KNOWN AND THE NEXT STEP
IS THE T BIO PROTEINS THE T
STAND FOR TARGETS AND THERE MAY
BE PUBLICATIONS AND MAY BE SOME
R01s AND THEN T CHECK WITH ONE
AT LEAST ONE BIO ACTIVE COMPOUND
AND THE LEAST STUDIED ARE THE
TCLIN PROTEINS THAT USUALLY HAVE
AT LEAST ONE APPROVED DRUG.
SO THIS IS ONE OF THE TOOLS
THAT'S INCLUDED IN PHAROS.
I CAN'T GO COMPREHENSIVELY OVER
ALL THE TOOLS IN PHAROS BUT I
WANTED TO HIGHLIGHT SOME TOOLS
THAT HAVE GONE THROUGH SOME
EALLITERATIONSS -- IL ITERATION
AND ON THE RIGHT YOU CAN SEE
THIS ALLOWS YOU TO SEE EXACTLY
WHERE YOUR PROTEIN EXPRESSED IN
THE BODY AND LIGHTER TISSUES ARE
LESS EXPRESSION AND WE HAVE THIS
AVAILABLE FOR REGIONS OF THE
BRAIN.
IF YOU ARE TRYING TO LOOK MORE
OF A CELL TYPE OR PARTICULAR
AREA OF A PARTICULAR TISSUE YOU
WANT TO SEE IF YOUR PROTEIN IS
EXPRESSED, THIS ALLOWS YOU TO
DRILL DOWN.
THIS IS THE CIRCLE PART.
IT ALLOWS YOU TO -- YOU CAN
START AT THE ORGAN LEVEL BUT IT
LETS YOU DRILL DOWN TO CELL TYPE
AND SEE WHERE YOUR PROTEIN IS
EXPRESSED.
A FEW OTHER NOTABLE FEATURES OF
PHAROS.
IF YOUR PROTEIN HAS APPROVED
DRUGS OR LIGANDS ARE LISTED AND
CLICK ON THEM AND IT WILL TAKE
YOU TO THE WEB PAGE TO LEARN
MORE ABOUT THE LIGANDS OR DRUGS
AND THERE'S A PROTEIN-PROTEIN
INTERACTION TOOLS AND SHOW OTHER
PROTEINS YOUR PARTICULAR PROTEIN
MAY INTERACT WITH.
YOU CAN CLICK AND TAKE YOU TO
THE WEB PAGE TO LEARN MORE.
ON THE RIGHT ARE DYNAMIC TOOLS
PHAROS.
THIS IS FROM ONE OF OUR CIT
AWARDEES PULLED FROM THE TOOL
CALLED REACTOME AND SEE WHERE
YOUR PROTEIN SITS IN TERMS OF A
PARTICULAR PATHWAY AND BUILDS IN
THE TARGET DEVELOPMENT LEVEL
INTO THAT.
YOU CAN SEE WHAT OTHER PROTEINS
YOU'RE INTERACTING WITH AND WHAT
WE KNOW ABOUT THEM.
IF IT'S A DARK PROTEIN IT WILL
PROVIDE PREDICTIVE PATHWAYS AS
WELL.
WE ALSO HAVE STRUCTURES BUILT
IN.
THE STRUCTURES IF KNOWN WILL BE
PROVIDED HOWEVER, WE HAVE
ALPOPOL IN THE INTERFACE.
IF THERE'S NOT A STRUCTURE IT
WILL PROVIDE PREDICTIVE
STRUCTURE.
IF IT'S AN IDG ELIGIBLE PROTEIN
YOU'LL SEE RESOURCES SUCH AS
CELL LINES, MOUSE MODELS,
ASSAYS, CONSTRUCTS.
THINGS ALONG THAT LINE.
SO JUST WANT IT GIVE YOU AN
IDEA.
THIS IS ESSENTIALLY A PRETTY
POPULAR TOOL.
STEADY USERSHIP OVER TIME AROUND
6,000 A MONTH.
WE ARE HAPPY IT SEE THIS IS
REMAINING POPULAR AMONG THE
SCIENTIFIC COMMUNITY.
AND THEN ON THE RIGHT IS REALLY
AN IDEA OF WHERE THINGS ARE
SHIFTING IN TERMS OF THOSE
TARGET DEVELOPMENT LEVELS.
IN 2018 THOSE ARE THE T DARK
PROTEINS TO REMIND YOU THE
BEGINNING OF THE IMPLEMENTATION
PHASE AND IN 2023, 50% FROM
MOVED TO THE T BIO OR T KIM
DESIGNATION.
THAT'S INDICATING THEY'RE
GETTING SOME LOVE AND ATTENTION
AND SEEING HOW THEY FIT INTO
HUMAN DISEASE.
NOT ALL THIS CAN BE ATTRIBUTED
TO JUST THE IDG PROGRAM BECAUSE
IT'S PART OF A MOVEMENT TO
ILLUMINATE THE STRUCTURE
PROTEINS AND OPEN TARGET AND
TARGET 25 AMONG OTHERS.
IT TELLS US WE'RE MAKING
PROGRESS IN BRINGING ATTENTION
TO THE UNDER STUDIED PROTEINS.
JUST AN OVERVIEW OF THE R3 PILOT
PROGRAMS AND THESE ARE 100,000S
CAN DIRECT COST AWARDS FOR ONE
YEAR.
-- $100,000.
WE WERE ABLE TO OBTAIN HIGH
LEVELS OF SCIENTIFIC OUTPUT FOR
LITTLE INVESTMENT.
THESE WILL SUPPORT THE
GENERATION OF PRELIMINARY DATA
AND TOOLS WITH PROTEINS IN THE
CONTEXT OF HUMAN DISEASE AND
WORKED ON DATA.
THE AWARDEES OBTAINED RESEARCH
RESOURCES FOR SUBSEQUENT LARGER
AWARDS FOR DRUG DISCOVERY
PROJECTS.
AND WE WERE ABLE TO FUND 9 8
AWARDS AND OVER 100 SPECIFIC
DARK PROTEINS VIA THE AWARDS
WITH OVER 100 PUBLICATIONS TO
DATE.
WE WERE ALSO ABLE TO FUND 29
EARLY STAGE OR NEW INVESTIGATORS
AND PULL IN EARLY STAGE
INVESTIGATORS, EARLY CAREER
INVESTIGATORS AND GET THEM
STUDYING THE PROTEINS AND BASED
ON DATA COLLECTED UNDER THE R3
PILOT AWARD PROJECT AWARDS.
AND SOME AWARDS ARE STILL ACTIVE
AND IT CAN TAKE TIME FOR
APPLICATIONS TO BE SUBMITTED AND
AWARDS TO BE MADE.
ONE PARTICULAR PROJECT THAT WENT
FROM PRETTY MUCH DARK UP TO
POTENTIAL CLINICAL TARGET IS
WORK DONE BY DR. STEVE LAUDNALL
AT THE UNIVERSITY OF MONTANA.
HE WORKED ON THE VIRAL IMMUNITY
AND INFLAMMATORY REACTION.
HE HAD A PRESENTATION AT THE
UNIVERSITY OF MONTANA AND THE
MAN ON THE BOTTOM HAS A SMALL
COMPANY AND PROPOSED WORKING
TOGETHER ON LOOKING AT AUTO
IMMUNE DISEASES SPECIFICALLY
CUTANEOUS LUPUS.
THEY WORKED WITH A SMALL COMPANY
AT THE TIME KNOWN AS VANT A.I.
AND THEY WORKED TO DESIGN
PROTEIN INHIBITORS AND THEY WERE
LOOKING FOR SOMEBODY TO GIVE
THEM ESSENTIALLY PROOF OF
CONCEPT STUDY TO TEST THEIR
INHIBITORS AND VALIDATE THEM AND
LOOK AT THE QUALITY.
THEY WERE ABLE TO OBTAIN THE
INHIBITORS FREE OF CHARGE.
AND IT WAS A COOL THING THAT
EMERGED DURING THE PARTNERSHIP.
AND THESE WERE REALLY THE FIRST
BINDERS THAT SHOWED SELECTIVITY.
SO THEY SUBMITTED AND WERE
AWARDED IN SBIR TO DESIGN SMALL
MOLECULES TO BLOCK FOR THE
TREATMENT OF CUTANEOUS LUPUS NOT
JUST IN ADULT BUT PEDIATRIC
PATIENT GROUPS AS WELL.
THEIR NEXT STEPS WILL BE IN VIVO
VALIDATION.
THEY WANT TO LOOK AT A ROLE FOR
RO3 IN ULCERATIVE COLITIS AS
WELL.
WITHIN COMMON FUND WE PARTNERED
WITH ANOTHER GROUP WORKING TO
MAKE ESSENTIALLY A GIANT
KNOCKOUT MOUSE RESOURCE.
THIS IS THE KNOCKOUT MOUSE
PHENOTYPING PROGRAM AND WE
INITIATED A PARTNERSHIP WITH
THEM IN SEPTEMBER OF 2022 TO TRY
TO DEVELOP 27 DIFFERENT UNDER
STUDIED ION CHANNEL KNOCKOUT
MOUSE AND IT'S IN PROGRESS BUT
THIS ONE IS ACTUALLY ONE OF THE
UNDER STUDIED ION CHANNELS A
PLLP AND THEY DO A LIMITED
PHENOTYPIC SUMMARY ON THE MOUSE
AND WHEN KNOCKED OUT HAS AN
EFFECT ON KIDNEY, TESTEES AND
PROVIDES THE SCIENTIFIC
COMMUNITY A STARTING POINT TO
UNDERSTAND WHERE THIS PARTICULAR
PROTEIN MAY BE INVOLVED IN HUMAN
DISEASE.
SO I'M GOING TO WRAP UP WITH ONE
LAST COOL THING WE'VE BEEN DOING
WITH THE GROUP IN ITALY.
SO THIS ACTUALLY A GROUP THAT IS
WITH THE ITALIAN GOVERNMENT AND
SAW VALUE IN THE INVESTMENT AND
THEY HAD A CALL RELEASED IN
ALLIANCE WITH RESPONSE TO A
GROWING NEED FOR TREATMENTS FOR
PATIENTS WHO SUFFER FROM RARE
DISEASE.
SO IT'S AN ITALIAN NON-PROFIT
ORGANIZATION WITH A FOCUS ON
FUNDING RESEARCH AND SUPPORTING
PATIENT GROUPS WITH RARE GENETIC
DISEASE AND FONDAZIONE IS IN THE
ORGANIZATION.
THE TWO OF THESE HAVE WORKED
TOGETHER TO HAVE SEVERAL FUNDING
CALLS AND I THINK IT'S THREE TO
DATE THAT UTILIZE PHAROS TO LOOK
AT T DARK PROTEINS WITHIN THE
CONTEXT OF RARE DISEASE AND
GENETIC AND NON-GENETIC ORIGIN.
THEY'VE BEEN ABLE TO PUT IN
ABOUT $13.4 MILLION EUROS AND
STUDY THE ROLE FOR T DARK
PROTEINS IN DIFFERENT AREAS OF
RARE DISEASE INCLUDING
MITOCHONDRIAL DISORDERS, MYELOID
NEOPLASMS AND NEUROLOGICAL
DISORDERS AMONG SEVERAL OTHERS.
THIS IS REALLY A NOTABLE EXAMPLE
OF HOW PHAROS AND THE STUDY OF
DARK PROTEINS CAN HELP MOVE THE
HUMAN DISEASE RESEARCH FORWARD.
AS CHRISTINE MENTIONED THE
COMMON FUND IS WRAPPING UP.
IT'S COMING TO AN END.
WE'RE AT THE END OF THE 10-YEAR
TIME LINE.
I WANT TO GIVE AN IDEA WHERE
THINGS SIT EITHER NOW OR IN THE
FUTURE.
SO BECAUSE AS YOU'VE SEEN THE
CAREER USEFULNESS OF PHAROS
THERE'S SEVERAL OPTIONS WE'RE
EXPLORING TO EXTEND IT AFTER THE
PROGRAM AND WE HAD GROUPS
UTILIZE IT AND USED FOR FUNDING
CALLS.
WE'RE GOING TO WORK HARD TO --
WE ARE WORKING HARD TO LOOK AT
DIFFERENT SUSTAINABILITY
INITIATIVES FOR PHAROS AND ON
THE RIGHT I WANT TO GIVE AN IDEA
WHERE ALL THE PROJECTS FALL.
THAT'S A WHEEL -- A CIRCLE PLOT
OF ALL THE DIFFERENT DISEASES
THAT WERE TOUCHED ON DURING THE
PROGRAM AND REALLY THAT PROGRAM
HAS ENCOURAGED OTHER GROUPS TO
TAKE ON THE FORMAT.
AND THERE'S AN A FORMAT TO OTHER
INITIATIVE.
WHAT IT DOES IS IT HELPS TO
INCORPORATE EARLY CAREER
INVESTIGATORS IN THE INITIATIVES
AND STIMULATE RESEARCH ON
INVESTMENT.
AND WE HAVE THE NCATS RARE
DISEASE NOFO WHICH HAS BEEN OUT
A COUPLE YEARS NOW AND WE LIKE
TO THANK COUNCIL FOR APPROVING
THE CONCEPT BECAUSE THAT HAS LAD
A TREMENDOUS IMPACT.
IN TERMS OF THE RESOURCES
DEVELOPED BY THE PROGRAM WE'RE
WORKING TO SUSTAIN THOSE THROUGH
THE UTILIZATION OF PUBLIC
REPOSITORIES AND OVER ALL WE
FEEL LIKE WE'VE HOPEFULLY
INCREASED INTEREST AROUND THE
UNDER STUDIED PROTEINS AND
REALLY DEMONSTRATED THE VALUE OF
LOOKING INTO THE PROTEINS IN
TERMS OF HUMAN DISEASE.
I'LL LEAVE YOU WITH THIS.
THIS IS A DRUGGABLE GENOME
SPECIAL SERIES WITH DRUG
DISCOVERY TODAY.
THE TOP PUBLICATION IS ONE THE
NIH PUT OUT AND THAT GOES OVER A
LOT OF THE OVERVIEWS IN TERMS OF
SUCCESSES AROUND THE PROGRAM AS
A WHOLE BUT THERE ARE A NUMBER
OF ADDITIONAL ARTICLES IN THE
SERIES THAT CAME OUT AND FROM
OUR SPECIFIC AWARDEES.
I'D ENCOURAGE YOU ALL TO TAKE A
LOOK AT THAT AND EXPLORE THE
SUCCESSES AND RESOURCES THE
PROGRAM PUT OUT.
SO I'M GOING TO HAND IT BACK TO
CHRISTINE.
>> BACK WITH A TEASER FOR NEXT
YEAR.
I'LL TRY TO GIVE A QUICK UPDATE
ON WHAT HAS HAPPENED TO LITCOIN
WITH AARON BERNSTEIN AND WAS
ONLY WITH US 10 MONTHS AND WAS
IMPRESSIVE AND TYLER BECK HAS
BEEN OVERSEEING THE PROGRAM AND
THE FOLKS YOU SEE NAMED ACROSS
THE BOTTOM ARE WORKING WITH US
THROUGH CONTRACT MECHANISMSS HEL
IS PAYING FOR AND THE BOTTOM
LINE IS THE IDEA IS TO CREATE
MACHINE READABLE, A.I. READY
KNOWLEDGE FROM LITERATURE.
WE CAN DO THIS PROSPECTIVELY
WITH NEWLY SUBMITTED MANUSCRIPTS
AND THIS IS WHAT WE HOPE TO DO
IN THE FUTURE.
ONE ASPECT IS WHAT WE REFER TO
AS A LITCOIN PUBLICATION FOR
MAT.
LITCOIN PROPER.
WE CAN DO THIS RETROSPECTIVELY
WORKING WITH THE ABSTRACT FROM
PUBLICATIONS ALREADY OUT AND
AVAILABLE.
WE'RE REFERRING TO THIS
RETROSPECTIVE PIECE AS LIT TO
GRAPH AND MAKING THE DISTINCTION
BETWEEN THE LIT COIN.
LIT2GRAPH GOES FROM AN ABSTRACT
HUMAN READABLE ABSTRACT TO
MACHINE READABLE GRAPHS.
YOU PROBABLY CAN'T READ THIS BUT
ON THE LEFT SIDE WE SEE AN
ABSTRACT.
ON THE RIGHT SIDE I WANT TO USE
THIS TO ILLUSTRATE TO COULD BE
VERT THIS TO A MACHINE READABLE
PIECE OF NFK.
WHAT'S THE NOVEL THING IN THE
ABSTRACT.
WHAT IS THE BOTTOM LINE OF THE
THING WE'RE TRYING TO PRESENT
AND WANT TO DO THAT COMPUTATION
AND IT'S TOO HARD FOR HUMANS TO
DO THIS.
IT'S TIME CONSUMING.
ON THE LEFT SIDE UNDER THAT
ABSTRACT I'M CIRCLING THIS
PARTICULAR STATEMENT.
ITS IS STATING HYPERTENSION DOES
NOT ABROGATE EDEMA AND SHOWS IT
WITH THE ARROW AND THE YELLOW
BOXES.
THE CURRENT STANDARD IS SEMMED.
IT'S BEEN AROUND SINCE 2008.
AT THE TIME IT WAS VERY CUTTING
EDGE.
IT WAS VERY FORWARD THINKING BUT
STRUGGLES IN A WAY TO GET THE
INFORMATION AND IT STRUGGLED TO
IDENTIFY WHAT IT THE POINT OF
THE PAPER AND WHAT IS BEING
RECORDED.
WE WANTED TO BENCHMARK AGAINST
SEMMED SINCE THAT'S THE
STANDARD.
ON THE RIGHT I'M SHOWING WHAT
AARON WAS ABLE TO DO IN 10
MONTHS WORKING WITH OUR
CONTRACTORS FOR LITCOIN AND THE
OTHER BENCHMARK YOU REALLY WANT
TO HAVE IS HUMAN.
HOW DOES WHAT WE DO WITH OUR S
COMPARE TO WHAT A HUMAN WOULD
ACTUALLY DO?
AND THERE'S THREE DIFFERENT
TRIPLES THAT GOT EXTRACTED FROM
THE SAME ABSTRACT AND ON THE
LEFT SIDE WITH THE SEMMED
THERE'S COLUMNS THAT SAY SUBJECT
CONTEXT, RELATION CONTEXT AND
BLANK.
ON THE RIGHT SIDE WE ARE GETTING
THE CONTEXT BEING IMPORTANT AND
TELLING YOU IN WHAT TISSUE THE
OBSERVATION WAS MADE OR IN WHAT
CELL TYPE OR IN WHAT POPULATION.
IT ENDS UP BEING VERY
SIGNIFICANT INFORMATION TO THE
TRIPLE THAT GETS EXTRACTED.
IF WE LOOK AT JUST NEXT TO WHERE
I HAVE CIRCLED HUMAN.
THE SUBJECT THE HUMANS
IDENTIFIED WAS HYPERTENSION.
WHEN I DO THIS WITH THE TOOL ON
WHICH WE'RE GOING TO BASE WHAT
WE CALL THE TOOL DOING THE
EXTRACTIONS IT COMES UP WITH
INDUCED HYPERTENSION WHICH IS
MORE ACCURATE.
SO THIS IS TOO EXCITING FOR ME
TO NOT TELL YOU ABOUT AND
HOPEFULLY IT MEANS EVEN IF
YOU'RE NOT GOING TO BE A COUNCIL
MEMBER NEXT YEAR YOU CAN TUNE IN
FOR THE UPDATE NEXT WE'RE.
SO WITH THAT I WILL CLOSE AND
KARLIE AND I HAVE AVAILABLE FOR
QUESTIONS.
>> ANY QUESTIONS FOR KARLIE OR
CHRISTINE?
>> THINK THE LITCOIN IS IMPR
IMPRESSIVE WE CAN HAVE A RICH
DATA SET AND BEING ABLE TO PULL
IN DATA FROM THE LITERATURE LIKE
LARGE LANGUAGE MODELS DO AND
WHAT WE'RE LOOKING AT IS TAKING
LARGE LANGUAGE MODELS TO
INTEGRATE ALL OF THESE DIFFERENT
DATA SETS TO BE QUERIABLE AND
ALL THAT IS A HUGE LEAP FORWARD.
WHEN DO YOU THINK YOU'LL BE
PUTTING IT OUT THERE?
>> UM, THAT'S A GOOD QUESTION WE
HAVE ANOTHER YEAR OF SUPPORT
FROM THE HEAL INITIATIVE.
DURING THAT TIME OUR GOAL IS TO
ACTUALLY WORK ON VERY FOCUSSED
LITERATURE THAT'S GOING TO BE
RELEVANT FOR PAIN AND SOME WITH
OUD.
AND WHAT WE WANT TO DO IS WORK
WITH THE RESEARCHERS TO BEGIN TO
SOCIALIZE THIS TO THE COMMUNITY
AND ALSO TO START TO TEST AND
FEEL OUT THE SYSTEM.
TYLER HAS BEEN BUSY TALKING WITH
PUBLISHERS AND EVERYTHING AND
WE'RE GETTING TRACTION THERE.
I DON'T KNOW IF YOU WANT TO COME
UP AND SAY A FEW WORDS BECAUSE
YOU'RE THE ONE WHO'S BEEN DOING
THE DAY TO DAY ON THIS.
>> SO, WITH THIS WE ARE PROBABLY
LOOKING TO SEE A FIRST HEAL
FOCUS GRAPH OF PAIN IN THE EARLY
SPRING.
AND THE GOAL IS TO GET AS MANY
HEAL INVESTIGATORS AS POSSIBLE
TO FINE TUNE THE GRAPH FROM THE
HEAL RESEARCHER AND RETRAINING
THE MODEL TO IMPROVE ITS
ACCURACY MORE BASED ON THAT
FEEDBACK AND THEN WE WOULD
EXPECT TO BE ABLE TO EXPAND TO
OTHER FIELDS AFTER THAT.
>> IS THAT A LOWER LIMIT IN
TERMS OF PERCENTAGE COVERAGE
THAT'S NEEDED TO MAKE THE LITHO
GRAPH USEFUL?
100%
100% COVERAGE IS NOT POSSIBLE
BUT IS 50% OR 60% DECENT TO GIVE
YOU ANSWERS?
>> I CAN TELL YOU RIGHT NOW I
THINK WE COULD MAKE USE OF THE
ALGORITHM BECAUSE THE DIFFERENCE
BETWEEN THE ACCURACY AND
CONTEXTUAL KNOWLEDGE IS SO VAST
WITH SOME OF THE PROJECTS.
THERE'S BEEN TOOLS FOR FINDING
RESULTS BUT CAN GO LEAPS AND
BOUNDS AHEAD ON THE VALUE BY
PULLING THIS KIND OF INFORMATION
EVEN IF IT WAS JUST FROM A
SINGLE SUB FIELD AT FIRST.
I DON'T THINK THERE'S A LOWER
LIMIT ON THAT REALLY.
>> AND YOU'RE ONLY LOOKING AT
PEER REVIEWED LITERATURE.
WE DON'T WANT TO NECESSARY START
LOOKING AT THINGS LIKE BIO
ARCHIVE UNTIL WE HAVE LAID THE
GROUND WORK FOR THE GRAPHS THAT
COME OUT OF AND FEEL CONFIDENCE
IN THE VALUE.
>> IF GRAPH-BASED SYSTEMS
WEREN'T ALREADY IN PLACE WE'D
TALKING ABOUT MEETING CRITICAL
MASS BUT THEY CAN GET INGESTED
AS WE GO AND SHOULD HAVE INSTANT
VALUE.
>> TALK ABOUT THE PROGRAM.
>> WE ARE WORKING WITH
SUBCONTRACTORS WORKING ON
GENERATING THE WEB INTERGRAPH
AND ABLE TO SHOW THE RESEARCHERS
WHO WELL EITHER OFFER OR
SOMETIMES ASSOCIATE THE WORK
FROM THE ORIGINAL ABSTRACT.
SHOW THEM WHAT THE OUTCOMES ARE
AND IF WE DO A GOOD JOB OR IF
THERE'S SOMETHING WE CKCAN EXTRT
BETTER AND USING THE
RELATIONSHIP.
USING THAT THEY'LL BE AUTOMOBILE
TOO -- ABLE TO GET THE FEEDBACK
AND IMPROVE IT ITERATIVELY.
>> THEY'LL WORK WITH BASICALLY
PUBLICATIONS THAT HAVE COME FROM
THE HEAL INVESTIGATOR LAB AND
WANT TO DO HACKATHONS WITH
POSTDOCS AND STUDENTS TO SAY
HEY, CAN YOU TAKE A LOOK AT THE
GRAPH ASSOCIATED WITH THE FAST
TRACK.
THAT INDIVIDUAL MAY NOT HAVE
BEEN AN AUTHOR BUT THEY'RE FROM
THAT LAB.
THEY SHOULD BE SOMEWHAT FAMILIAR
AND BE ABLE TO TELL US HOW WE'RE
DOING AND I THINK AS WE GO ALONG
THE ARLING ALGORITHM WILL GET
BETTER AND BETTER.
>> THE LONGER TERM EXPECTATION
IS WE HAVE A TRUE WEB INTERFACE
WHERE ANY RESEARCHER COULD DO,
FIND THE PAPERS THEY WERE
ASSOCIATED WITH AND THEY HELPED
PUBLISH AND DO THE CURATION ON
WHATEVER RESEARCH THEY PUBLISHED
TO THEN FEEDBACK INTO THE
SYSTEM.
>> THAT'S GREAT.
>> I MADE A COMMENT HOW YOU HAVE
ANOTHER YEAR OF FUNDING ON THIS.
IT SOUNDS LIKE IT HAS THE
POTENTIAL FOR BEING ALMOST A
WIKIPEDIA LIKE OPEN SOURCE.
I DON'T MEAN WIKIPEDIA BUT THE
CONCEPT OF THE COMMUNITY
ENRICHED AND IMPROVE.
DID I HEAR A HINT OF THE
FUNDING'S GOING AWAY?
IT'S SUCH AN EXCITING PROJECT
AND IT'S WONDERFUL TO WATCH IT
EVOLVE OVER THE PAST COUPLE
YEARS.
>> IT'S PAINFUL TO HAVE THIS
HAPPEN AT A TIME WHEN THE NIH IS
FACING A TIME WHEN WE'RE GOING
TO BE REALLY STRUGGLING FOR
FUNDING.
THERE'S BEEN SO MUCH IS INTEREST
AND ENTHUSIASM.
I'M FEELING OPTIMISTIC WE'LL BE
ABLE TO FIND A FUNDING SOURCE TO
KEEP THIS GOING AND TO BUILD IT
OUT FURTHER.
I HAVE TO MENTION WE PRESENTED
THIS TO DR. LANGEVIN THE HEAD OF
NCCIH AND HAD AN IDEA OF
INCORPORATING TEXTBOOK KNOWLEDGE
INTO THIS.
IF WE DO THAT THEN THE POWER IS
BLOWN OUT OF THE WATER.
WHAT WE DO WITH SCIENTIFIC
LITERATURE IS GOING TO GET
MARRIED WITH HUMAN USER'S
KNOWLEDGE.
AND IF WE CAN INCORPORATE
TEXTBOOK KNOWLEDGE IT WILL BE
PHENOMENALLY POWERFUL.
AND THE RATE AT WHICH WE CAN
ACCELERATE RESEARCH -- I
ANTICIPATE IT WILL BE LIKE
NOTHING WE'VE SEEN BEFORE BUT
I'M PROBABLY BIASSED.
>> ANY OTHER QUESTIONS?
THANK YOU, CHRISTINE AND KARLIE.
I THINK WE'RE NOW TRANSITIONING
TO OUR CONCEPT CLEARANCES.
THERE'S A VARIETY FOR EACH
CONCEPT CLEARANCE.
CHRISTINE HASN'T MOVED BECAUSE
SHE'S GOING TO GO FIRST.
SO CHRISTINE, GO AHEAD AND KICK
OFF THE CONCEPT.
>> THANK YOU.
AS PROMISED, THE WHY UPDATE WAS
ON THE FIRST PRONG AND NOW WE'LL
TALK ABOUT THE SECOND PRONG.
I'VE SHOWED YOU THIS AND I'LL
BLOW PAST IT BECAUSE WE TALKED
ABOUT IT ALREADY.
BASICALLY WE HAVE VARIOUS THINGS
WE'RE ARE USING EXPLORING
DRUGGABLE PROTEINS AS WELL AS
MODULATING TARGETS IS WHAT
YOU'LL HEAR ABOUT NOW.
AND TRYING TO CAPITALIZE ON
SPEED AND A.I.
SO THIS IS A CONCEPT CLEARANCE
FOR EXPANDING THE TARGET
LANDSCAPE BY DRUGGING THE
UNDRUGGABLE.
THIS IS SOMETHING THAT'S THE
DRUG DISCOVERY WHEEL AND RELIES
ON SMALL MOLECULES AN BIOLOGICS
AND WHAT IT MEANS IS THAT YOU
FIND A TARGET THAT'S RELATED TO
HUMAN DISEASE AND USUALLY
PROTEIN AND GENERATE WAYS
AGAINST THE TARGET AND OPTIMIZED
AND TAKE THEM TO THE PRE
CLINICAL STAGE.
HOPEFULLY TO THE CLINICAL STAGE
AND IN THEORY GET TO A DRUG.
THE PROBLEM IS THEY'RE CAUSED BY
GENES AND AN INTRACTABLE DISEASE
IS UNABLE TO BE RELIEVED OR
CURED THROUGH CURRENT THERAPIES.
10% ARE DRUGGABLE AND 5% IS
DRUGGABLE AND DISEASE RELEVANT.
FROM THE PROTEINS ON THE RIGHT
ABOUT 60% ARE BELIEVED TO BE
UNDRUGGABLE.
WE DON'T HAVE A WHOLE LOT OF
DRUGS.
MAYBE 13% HAVE LAUNCH DRUGS.
THERE'S A LOT TO EXPLORE.
THE CLASSICALLY DRUGGABLE
TARGETS ARE ABLE TO FIND
LIGANDS.
THOSE ARE THINGS LIKE RECEPTORS
AND ENZYME.
THERE'S AN ENTIRE GROUP OF
PROTEINS THAT CAUSE HUMAN
DISEASE AND FALL IN THE DISEASE
CATEGORY CAUSED BY THE
PROTEIN-PROTEIN INTERACTIONS AND
CAN BE CAUSED BY PROTEINS OR
OTHER PROTEINS THAT DON'T HAVE
BINDING POCKETS.
NOW, OUTSIDE OF THE PROTEINS WE
KNOW A NUMBER OF DISEASES CAUSED
DIRECTLY BY RNA.
AND SO THAT'S ANOTHER
OPPORTUNITY IN TERMS OF ONE OF
THESE HOPEFULLY TARGETS WE CAN
IDENTIFY AND TAKE TA FORWARD
FOR TREATMENT AND WE CAN
ESSENTIALLY IDENTIFY A WAY OF
DRUGGING UPSTREAM SOME
UNDRUGGABLE PROTEIN TARGETS.
FINALLY I WANT TO HIGHLIGHT
MOLECULAR ENTITIES INCLUDING
LIPIDS AND METABOLITES AND
ORGANELLES
AND AND THES IS JUST A SMALL
SNAPSHOT BUT THERE'S DISEASES
CAUSED BY UNDRUGGABLE TARGETS
AND MANY THAT ARE ZEETZS CAUSED
DIRECTLY BY RNA EXPANSIONS AND
THERE'S NEUROLOGICAL DISEASES
LIKE ALZHEIMER'S DISEASE AND
PARKINSON'S CAUSED BY PROTEINS
AND BUILD UP OF THESE PLAQUES.
AND THE DISEASES SUCH AS CANCER
NEURO PATHIC PAIN AND INFECTIONS
AND OTHERS CAUSED BY OTHER
ENTITIES LIKE PATHOLOGICAL
LITHOGRAPHS AND THERE'S BEEN
KRAS.
IT WAS THOUGHT TO BE UNDRUGGABLE
BUT HE WAS ABLE TO IDENTIFY A
WAY TO EDIT AND IT'S PROMISING
IN THE WAY OF TUMORS.
THERE'S BEEN WORK TOWARDS
DEGRADING RNA AND THAT CAN BE A
GAME CHANGER IN CONTRIBUTORS AND
ALSO DISEASES WHERE PROTEINS
CANNOT BE TARGET AND THE RNA CAN
BE TARGETED INSTEAD.
AT THE BOTTOM THEY IDENTIFY A
WAY TO STABILIZE PROTEINS SUCH
AS IN CYSTIC FIBROSIS AND WHERE
ARE OPPORTUNITIES IN IDENTIFYING
NEW TARGETS AND NEW PLATFORMS
AND MODALITIES WE COULD LEVERAGE
TO DRUG UNDRUGGABLE TARGETS.
THIS WAS THE QUEST FOR TREATMENT
MODALITIES FOR INTRACTABLE
DISEASE TARGETS IN COLLABORATION
WITH NCI, NINDS AND NIAID.
WE WORKED TO IDENTIFY FAMILIES
IN TERMS OF THERAPEUTIC
MODALITIES AND NEW OR EMERGING
TECHNOLOGIES THAT COULD BE
LEVERAGED TO ADDRESS LIMITATIONS
OF TREATMENT MODALITIES.
AND PREVENT OR SLOW THE
DEVELOPMENT OF STRATEGIES FOR
TREATING HUMAN DISEASES
ASSOCIATED WITH THE UNDRUGGABLE
TARGETS.
AS A RESULT CAN BE SEEN HERE.
WE PUBLISHED A PAPER IN DRUG
DISCOVERY TODAY.
IF YOU'RE CURIOUS TO LEARN WHAT
ALL THE RESULTS OF THE WORKSHOP
WERE, TAKE A LOOK AT THE PAPER.
WHAT IT TOLD US IS THAT THE
FIELD IS PRIME FOR NEW CLASSES
OF ME MODALITIES AND TARGETS.
SO NCATS LOOKS AT NOVEL TARGET
CLASSES TO TREAT HUMAN DISEASE.
THE OBJECTIVE IS TO SUPPORT PRE
CLINICAL DEVELOPMENT OF
TREATMENTS FOR DISEASES FOR
PREVIOUSLY UNDRUGGABLE TARGETS
AND RNAs AND PROTEINS AND THINGS
OF THAT NATURE AND THIS IS A NEW
INITIATIVE THAT WOULD FILL GAPS
AND RELIEF PRESSURE POINTS
PREVENTING OR SLOWING THE
DEVELOPMENT OF TREATMENT FOR
INTRACTABLE HUMAN DISEASES AB
THOSE ASSOCIATED WITH
UNTREATABLE DRUGGABLE TARGETS
AND HOPE IT PROVES MODULATION OF
A PREVIOUSLY UNDRUGGABLE TARGETS
AND BRING IN IDEAS HOW TO DRUG
AN UNDRUGGABLE TARGET CLASS.
THERE ARE ASPECTS OF THE
INITIATIVE.
IT FILLS THE NEED FOR AN
INITIATIVE ON ENCOURAGING THE
SCIENTIFIC COMMUNITY TO EXPLORE
NOVEL UNCONVENTIONAL RESEARCH
STRATEGIES TO EXPAND THE
THERAPEUTIC TARGETS AND
DISEASES.
AND ENCOURAGES SIGNIFICANT
PROCESS IMPROVEMENT FOR
IDENTIFYING NOVEL TARGETS FOR
DISEASE AND OPENS NEW AREAS OF
RESEARCH IN DRUG DISCOVERY
MODALITIES
AND WHAT WE'RE WORKING TOWARDS
IS ADDRESSING PATIENT PONGSES.
POPULATIONS.
WE EXPECTS IT WILL ADDRESS
CLASSES WITH PATIENT GROUPS OF
UNMET NEEDS AND PROVIDE PROOF OF
CONCEPT STUDIES AND SPUR
INNOVATION IN THE NEW TREATMENTS
AND TREAT THOSE WHO SUFFER FROM
THESE INTRACTABLE DISEASES.
IN SUMMARY WE'RE WORKING TO
SUPPORT PRECLINICAL DEVELOPMENT
FOR TREATMENTS OF DISEASES WITH
TRADITIONALLY UNDRUGGABLE
TARGETS AND NEURO DEGENERATIVE
DISEASE AND MITOCHONDRIAL
DISEASES AND PAIN AND THERE'S
MORE I HAVE NOT MENTIONED.
THERE'S A NEED TO ADDRESS THE
DISEASE GROUPS BECAUSE THERE'S
LITTLE TO NO THERAPEUTIC
DEVELOPMENT DUE TO THE UND
UNDRUGABILITY OF THE CLASSES.
WE'RE LOOKING TO EXPAND THE
GROUP AND INCLUDING PROTEIN
DEGRADATION METHODS AND
STABILIZATION TECHNOLOGIES,
MOLECULES AND MINI PROTEINS.
WE'RE EXPANDING SOME OF THE
BASIC DRUG DISCOVERY PLATFORMS.
WHAT MIGHT IMPROVE THE
INITIATIVE AND WHAT ARE KEY
POINTS TO CONSIDER AND WHAT DOES
SUCCESS LOOK LIKE BUT WE HAVE A
COUPLE SPECIFIC ONES TOO.
WHAT OTHER INTRACTABLE DISEASE
GROUPS SHOULD WE BE CONSIDERING
AND SHOULD WE CONSIDER WORKING
WITH BIO FRAR PHARMA AND WELCOME
ANY OTHER QUESTIONS TOO.
>> SUCCESS IS SOMETHING KARLIE
LEADS.
WHILE I HAVE NOTHING TO RAD BUT
WHILE YOU WERE SPEAKING ABOUT
WHAT IS THE GAME PLAN TO WORK
WITH THE PHARMACEUTICAL INDUSTRY
TO IMPLEMENT AT SCALE.
YOU
>> THE IDEA IS TO TRY TO GET THE
PLATFORMS AND SHOW THE PROOF OF
CONCEPTS TO GO TO PHARMA OR
SMALL COMPANIES OR WHOEVER AND
TELL THEM THEY WORK.
THREES A CLEAR PATHWAY HERE ALSO
IN TERMS OF SETTING UP THE
PLATFORM AND GOING TO SBIR.
THAT'S ALSO A WAY TO GET THIS
STUFF OFF THE GROUND.
>> GREAT.
FOR ME, PERSPECTIVE OF THE
INTRACTABLE DISEASE GROUP IT'S
INTERESTING YOU HIGHLIGHTED
DISEASES OF AGING.
BETWEEN COVERING THOSE MASSIVE
DISEASES OF AGING AND THINKING
ABOUT THE NEURAL DEVELOPMENT AT
OTHER END OF THE LIFE SPAN WHICH
INCORPORATES SO MANY OF THE RARE
DISEASE CATEGORIES WHERE
FAMILIES SUFFER.
>> GOT IT.
>> I HAVE NO OTHER COMMENTS.
WE HAVE TWO OTHER DISCUSSANTS.
>> THANK YOU.
FABULOUS JOB.
THAT WAS AWESOME.
GETTING THIS READY TO HEAR ANDS
SHOWING A GREAT MODEL FOR
SUCCESS.
GREAT JOB.
WHAT IF IF YOU USED THE TOOL WE
PRESENTED TO US EARLIER AS A WAY
OF INTEGRATING AND EVALUATING
AND I LOVE THE IDEA OF THE
DEVELOPMENT LIFE SPAN
PERSPECTIVE BUT WHAT IF YOU USE
THAT AS WELL AND LOOKED AMOUNT
THAT INTERSECTION PERHAPS?
>> WE COULD DO THAT.
ONE GREAT THING ABOUT THE
CONCEPT IS THERE'S A LOT OF
DIRECTIONS TO GO.
THERE'S UNDRUGGABLE TARGETS AND
WAYS TO LOOK AT THOSE.
I MADE A NOTE.
WE'LL CHECK IT OUT.
>> THAT'S AN EXCELLENT
PRESENTATION.
I DO THINK THE PLATFORMS ARE
CRITICAL.
WE DO METABOLOMICS AND
PROTEOMICS AND SINGLE CELL
ANALYSIS AND ALL OF THAT.
BEING ABLE TO HAVE A PLATFORM
WHERE SOMEONE DOESN'T VA TO BE
AN EXPERT IN DOING THAT BUT CAN
HAVE A GREAT IDEA AND MOVING IT
FORWARD.
WE SAW THAT WITH THE COVID
VACCINE.
WE'VE SEEN THIS WITH DIFFERENT
ASPECTS.
YOU'RE GOING TO FIND I THINK
WE'LL LOOK DEEPER AT THE STUFF
WE JUST TALKED ABOUT JUST
SCRATCHES THE SURFACE OF WHAT
WOULD BE DRUGGABLE IN TREATING A
DISEASE.
I DO THINK A KEY ASPECT OF THIS
IS THE PLATFORMS FOR DOING THIS
STUDIES AND TESTING IT AND MASS,
IF YOU WILL, BEING ABLE TOO RUN
THROUGH A LIPID PLATFORM,
RUNNING THROUGH LIPIDS AND VICE
VERSA FOR PRETTY MUCH ANY OTHER
TARGET YOU CAN THINK OF.
>> I WILL MENTION ONE OF THE
OUTCOMES WAS ABOUT PLATFORMS AND
THE OBSERVATION WAS WITH THE
DRUG DISCOVERY -- THE REASON I
SHOWED THAT WHEEL IS A LOT OF
THE PLATFORMS BUILT INTO THE
DRUG DISCOVERY ODYSSEY NOW ARE
SPECIFICALLY FOCUSSED ON A SMALL
MOLECULE OR BIOLOGIC
DEVELOPMENT.
THAT MEANS THERE'S NOT A LOT OF
OPPORTUNITIES IN TERMS OF
GETTING WAYS IN IN TERMS OF
OTHER TARGET CLASSES THAT MAY
NOT FALL INTO A BINDING POCKET
OR A WAY A BIOLOGY CAN ATTENUATE
IT.
YEAH.
>> ONE MORE COMMENT BECAUSE IT
GETS BACK TO THE LITCOIN, A.I.,
YOU CAN'T ALWAYS TRUST IT.
IN FACT OFTEN YOU CAN'T TRUST
IT.
IT WILL COME UP WITH THINGS NO
ONE IN OUR RIGHT MINDS OR EVEN
WRONG MINDS WOULD THINK OF,
RIGHT.
SO SOME OF THE OBSERVATIONS THAT
WILL COME OUT A LOT OF THEM WILL
BE CRAZY AND WRONG BUT THE VOICE
IN THE WILDERNESS IS THE STUFF
OF NOBEL PRIZES.
ULCERS BEING CAUSED BY A
BACTERIA, THAT'S STUPID.
WELL, GUESS WHAT, IT'S NOT.
WE HAVE TO BE CAREFUL NOT TO GO
DOWN TOO MANY WRONG PATHWAYS BUT
INTERESTED TO SEE HOW TO MERGE
LITCOIN WITH ALSO THIS
INITIATIVE.
>> ANY OTHER COMMENTS OR
QUESTIONS?
>> THANK YOU FOR THE
PRESENTATIONS.
GREAT.
JUST THINKING ABOUT THE
INTRODUCTION TO THIS IN IDG,
THAT SPEAKS FOR POTENTIAL
SACRIFICE FOR A PROGRAM.
I'M NOT SURE YOU ANTICIPATED NOT
JUST WHAT WOULD BE DRUGGED BUT
HOW THEY'LL BE DRUGGED.
THEY DIDN'T EVEN REALLY EXIST
WHEN IDG WAS LAUNCHED.
YOU GAVE AN EXAMPLE AND I KNOW
PEOPLE WHO A TARGET FOR RARE
DISEASE AND WOULD LOVE A
MOLECULE AND YOU USED IT AS AN
EXAMPLE AND WE HAVE TO CONNECT
THEM UP.
>> YEAH.
>> JUST IN THE LAST HOUR.
I THINK IT'S EXCITING YOUR
EXPANDING.
YOU KNOW HOW I THINK ABOUT
UNDRUGGABLE AND THIS MAKES THEM
DRUGS.
>> WE'RE EXCITED.
I THINK IT'S ONE OF THOSE THINGS
IT'S LIKE AN OLD-NEW TECHNOLOGY.
I SAY THAT BECAUSE IT'S CUTTING
EDGE AND BECOMING OLD IN THE
SENSE IT'S BEEN AROUND FOR A
WHILE.
THEY'RE CONSTANTLY COMING UP
WITH NEW WAYS TO USE IT AND IT'S
NOT CALLED PROTAX BUT IT'S A
SIMILAR SYSTEM USED FOR
DEGRADING RNA.
THE OTHER GROUP I SHOWED YOU,
DAN AND LYDIA THEIR TECHNOLOGY
IS ALSO RELATED AND RATHER THAN
DEGRADING THE PROTEINS,
STABILIZES THE PROTEIN.
IT'S WAYS TO USE SOMETHING WE
ALREADY HAVE.
PROTAC IT ALMOST CAME OUT OF
NOWHERE AND HAS BEEN USEFUL AND
HOPING THOSE WILL BE THE TYPES
OF THINGS TO STIMULATE.
>> I THINK IT'S POSSIBLE WITH
WHAT YOU OUTLINE IF YOUR PAST
SUCCESS IS PREDICTIVE OF YOUR
FUTURE SUCCESS YOU MAY END UP
WITH A RATE OF DISCOVERY SO FAR
OUTPACED BY THE FUNDING
MECHANISMS AND IT'S GOING TO BE
SOMETHING INSTITUTIONAL TO THINK
ABOUT.
IF THE MISSION IS TO GET MORE
THINGS TO MORE PEOPLE THE OLD
WAY -- HOW DO YOU SHRINK THE
FUNDING CYCLES?
THIS REALLY IS AN ACCELERATOR
AND YOUR OTHER PREDICTIONS IS
MORE IS TO COME.
A NOTE OF RISK AND TO THINK
ABOUT THE FUNDING.
>> YEP.
YEP.
DEFINITELY.
>> THANK YOU.
AND I LOVE THE RECOMMENDATIONS
TO SYNERGIZE.
>> IF THERE'S NO FURTHER
DISCUSSION COULD WE HAVE A
MOTION TO APPROVE THE CONCEPT.
SECOND.
ALL IN FAVOR.
ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT THE CONCEPT IS
APPROVED.
>> THANK YOU ALL.
>> THANK YOU.
THE NEXT CONCEPT WE HAVE TWO.
THIS IS THE SECOND OF THREE
MOVING ON TO THE OFFICE OF
SPECIAL INITIATIVES WITH DAN
TAGLE.
>> I'M THE DIRECTOR FOR THE
OFFICE OF SPECIAL INITIATIVES
AND TELL YOU MORE ABOUT OUR
OFFICE.
OUR OFFICE MISSION IS TO ADDRESS
TRANSLATIONAL PROBLEMS WITHIN
INNOVATIVE SOLUTIONS THROUGH
DISRUPTIVE TECHNOLOGIES.
WE DO THIS BY NOVEL
PARTNERSHIPS.
OUR ANTICIPATED END GOAL IS TO
CREATE A PARADIGM SHIFT IN THE
FIELD.
6:
SO, WHAT WE HAVE BEEN DOING OVER
THE PAST 10 YEARS THE FUND
RESEARCH TO ACCEPTANCE ADHERING
TO THE PRINCIPLES OF DEVELOPING
THE TECHNOLOGY, DEMONSTRATING
THE UTILITY IN RESEARCH AND THEN
EVENTUALLY DISSEMINATING IT FOR
USE BY THE BIOMEDICAL RESEARCH
COMMUNITY.
YOU CAN SEE HERE A NUMBER OF
DIFFERENT PROGRAMS THAT WE HAVE
AT VARIOUS STAGES.
WE ARE GRATEFUL TO COUNCIL AT
THE LAST COUNCIL ROUND FOR
APPROVING OUR QUANTUM
TECHNOLOGIES YOU CAN SEE IN PINK
IS ONE OF OUR YOUNGEST PROGRAMS
AND WE'RE ABOUT TO LARGE A PRICE
COMPETITION AT THE END OF THE
MONTH ON QUANTUM TECHNOLOGIES.
WHAT THE PROPOSED CONCEPT THIS
AFTERNOON IS ALL ABOUT, TWO
TECHNOLOGIES.
TWO EXCITING AREAS.
ONE CALLED EXTREME OR EXOSOME
THERAPY AND EXTRA CELLULAR RNA.
THIS IS A COMMON FUND PROGRAM
THAT NCATS IS LEADING.
IT'S BEEN USEFUL IN ELUCIDATING
HOW EXTRA CELLULAR RNA WITH THE
VESICLES ARE BEING USED BY OUR
CELLS FOR INTRACELLULAR
COMMUNICATION.
THAT'S A PARADIGM SHIFT IN HOW
WE THOUGHT ABOUT COMMUNICATION
WITHIN CELLS.
THEN THE OTHER PROGRAM AS BEING
USEFUL TOOLS TO REPRESENT HUMAN
PHYSIOLOGY.
THE CONCEPT IS REALLY AN
EXCITING AND PROMISING MERGER OF
THE TWO TECHNOLOGIES IN SOME
WAYS IT'S A SYNERGISM OF THE TWO
TECHNOLOGIES.
AND LEADING TO A MUCH MORE
POWERFUL APPROACH.
WITH THAT IT WILL BE PRESENTED
BY CHRISTINE HAPPEL THE PROGRAM
OFFICER THAT LEADS THE EXTRA
CELLULAR RNA PROGRAM AND
FOLLOWED BY DR. HARGROVE-GRIMES
WHO LEADS A CLINICAL TRIAL.
WITH THAT I'LL TURN IT OVER TO
CHRISTINE.
THANK YOU.
SO MY NAME IS CHRISTINE HAPPEL.
IN COLLABORATIVE SPIRIT I'LL BE
TAG TEAMING WITH MY COLLEAGUE,
DR. PASSLY HARGROVE-GRIMES.
I'LL TALK ABOUT SELECTIVE
PRECISION TARGETING.
NCATS SPENT YEARS LEADING
TISSUES ON A CHIP.
IN ADDITION TO LEADING MULTIPLE
PROGRAM ON EXTRA CELLULAR
COMMUNICATION.
WE NOW AIM TO CAPITALIZE ON THE
DATA WE HAVE DEVELOPED IN THE
PROGRAMS AND MERGE THE AREAS TO
ADDRESS A MAJOR CHALLENGE.
AND THAT CHALLENGE IS THAT SOME
INTERVENTIONS REQUIRE THE SAFE
AND EFFECTIVE DELIVERY OF
MOLECULES TO SPECIFIC CELLS AND
TISSUES.
NOW THESE CAN INCLUDE THINGS
LIKE GENE EDITING FOR MUSCULAR
DYSTROPHY.
IT COULD INCLUDE MONOCLONAL
ANTIBODIES OR INTERVENTIONS THAT
REGULATE GENE EXPRESSION FOR A
GROWING NUMBER OF PATHOLOGIES
FROM CANCER TO NEURODEGENERATIVE
DISEASES.
WE PROPOSE TARGETED DRUG
DELIVERY SYSTEMS.
WHAT'S THAT MEAN?
TARGETED DRUG DELIVERY SYSTEMS
ARE A METHOD OF DELIVERING
MEDICATIONS TO A SPECIFIC AREA
OF THE BODY.
SO THIS COULD BE AN ORGAN, A
TISSUE OR CELL TO INCREASE THE
CONCENTRATION OF THE DRUG IN
THAT PARTICULAR AREA.
SO WITH THAT WE'RE PROPOSING THE
CONCEPT COLLECTIVE PRECISION
TARGETING TOO DETERMINE THE
MECHANISM FOR PRECISE ON TARGET
THERAPEUTIC DRUG DELIVERY TO
SPECIFIC HARD TO REACH LOCATIONS
USING NPS.
SO WE'RE TRYING TO DELIVER
THERAPEUTICS TO THE RIGHT TISSUE
ADDRESS.
NOW TARGETED DRUG DELIVERY
SYSTEMS COULD INCLUDE NANO
PARTI
PARTICLES, SYNTHETIC OR THINGS
LIKE ANTIBODIES, CARRIERS AND
TECHNOLOGIES SUCH AS FOCUSSED
ULTRASOUND.
SO FOCUSSED ULTRASOUND IS A
TECHNOLOGY THAT GUIDES
THERAPEUTIC TO SPECIFIC
LOCATIONS AND CAN ACROSS THE
BLOOD BRAIN BARRIER.
THE MODALITIES SHOULD BE BIO
COMPATIBLE AND BIOLOGICAL
BARRIERS AND TARGET A SPECIFIC
CELL TISSUE OR ORGAN OF
INTEREST.
WE THINK THE MULTI-ORGAN NPS CAN
SERVE AS A VALUABLE TOOL FOR
THIS ENDEAVOR.
SO WHAT ARE MICROPHYSIOLOGICAL
SYSTEMS.
THEY'RE TISSUE CHIPS OR BIO
ENGINEERED DEVICES THE SIZE OF A
USB THUMB DRIVE TO GROW HUMAN
CELLS AND TISSUES AND TEST THEM
FOR TARGETING WITH SAFETY,
EFFICACY AND TOXICITY
ASSESSMENTS.
THEY CONTAIN CELL TYPES AND MORE
PREDICTIVE OF IN VIVO LIKE
BIOLOGY THAN TRADITIONAL MODELS.
YOU CAN SEE AN EXAMPLE OF A
HUMAN MULTI-ORGAN SYSTEM FOR
HEART, BLOOD BRAIN BARRIER AND
SKIN.
ONE INTEGRATED THE FLOW ALLOWS
TISSUE COMMUNICATION ACROSS
DIVERSE BIOLOGICAL BARRIERS.
YOU CAN SEE AN INLET AREA TO ADD
OUR THERAPEUTIC MODALITY OF
CHOICE.
NOW, I WANT TO BE CLEAR THIS IS
JUST AN EXAMPLE AND THIS IS NOT
THE FULL GAMUT OF WHAT WE COULD
TEST.
WE WERE INTERESTED
IMMUNOGENICITY AND NEED THE
IMMUNE COMPONENT.
IF WE WERE INTERESTED IN
MODELLING WOULD BE THE LIVER
COMPONENT TO ADDRESS BIO
DISTRIBUTION OR VASCULATURE TO
ADDRESS SYSTEMIC ADMINISTRATION
THEN GOAL IS TO USE THE HIGHLY
PREDICTIVE MICROORGAN SYSTEMS TO
PROVIDE EVIDENCE OF THERAPEUTIC
TARGETING TO HARD TO REACH
LOCATIONS WITHIN THE HUMAN BODY
AND MAY INCLUDE RETINA, BLOOD
BRAIN BARRIER, CNS TISSUE AND
PLACENTA AND WE'LL TARGET THE
SYSTEMS USING A BROAD SPECTRUM
OF THERAPEUTIC CARGO, DRUGS, SN
RNAs AND GENE EDITING TOOLS.
WE HAVE FOUR GOALS TARGET TO THE
ORGAN OR CELL OF INTEREST.
ASSESS OFF TARGET AFFECTS IN THE
SYSTEM AND EVIDENCE OF CARGO
ACTIVITY AND FOURTH IS TO
DETERMINE THE OPTIMAL ROOT OF
ADMINISTRATION.
METRICS FOR SUCCESS WILL INCLUDE
THE ADMINISTRATION OF THE
ABILITY TO PRECISELY TARGET A
CELL OR TISSUE TYPE OF INTEREST
USING THE SYSTEMS.
RESEARCH ACTIVITIES AND RESULTS
ARE EXPECTED TO ENABLE
TREATMENTS FOR HARD TO TREAT
DISEASES SUCH AS PRETERM BIRTH
WHICH CURRENTLY HAS NO CURRENT
TREATMENTS AND HOW DO WE GET
MOW -- MODALITIES TO THE
PLACENTA AND A DISEASE CONTEXT.
SO TO SUMMARIZE TARGETING DRUG
DELIVERY PLATFORMS ARE APP
APPLICABLE TO DISEASES AN
CONDITIONS.
THEY AFFECT SAFETY AND TOXICITY
AND CAN ALSO IMPROVE EFFICACY AS
WELL AS DOSING AND ADDRESS OFF
TARGET EFFECTS.
THERE'S REALLY MANY ADVANTAGES.
AND IN SUMMARY, WE ARE POSING
SELECTIVE PRECISION TARGETING AS
THE END GOAL OF THE PROGRAM TO
ENABLE PRECISE ON TARGET DRUG
DELIVERY TO SPECIFIC HARD TO
REACH LOCATIONS WITHIN THE HUMAN
BODY USING MULTIORGAN NPF AND
DELIVER TO THE RIGHT TISSUE
ADDRESS.
SO WITH THAT, WE DO HAVE A FEW
QUESTIONS WE'D LIKE TO ADDRESS
TO COUNCIL.
FIRST, WHAT MIGHT IMPROVE THE
INITIATIVE AND WHAT ARE KEY
POINTS FOR US TO CONSIDER.
AND THEN SPECIFICALLY WHO ARE
POTENTIAL PARTNERS OR
STAKEHOLDERS THAT SHOULD BE
INVOLVED?
WITH THAT, THANK YOU AND WE'LL
BE HAPPY TO ACHE ANY QUESTIONS.
>> THANK YOU.
THAT WAS VERY GOOD.
SO WE'LL HAVE OUR THREE
DISCUSSANTS.
PAUL.
>> FIRST A QUESTION.
DO YOU HAVE PILOT DATA THAT SHOW
WHEN YOU DELIVER THESE
INTERVENTIONS TO MULTI-CELL
TISSUE ON CHIP ECO SYSTEM THAT
THOSE CELLS BEHAVE DIFFERENTLY?
CAN YOU REPHRASE YOUR QUESTION,
I'M SORRY.
THEY BEHAVE DIFFERENTLY THAN IN
VIVO.
>> YOU'VE DONE THE WORK WITH
SINGLE CHIP.
THE NOVELTY WHAT I'M HEARING IS
YOU'RE NOW CREATING MULTI-CHIP
TO SIMULATE A MORE COMPLEX IN
VIVO ENVIRONMENT.
THE DIFFERENT REACTION TO THOSE
INTERVENTIONS.
HAVE YOU SHOWN IT MAKES A
DIFFERENCE WHEN YOU COMBINE THE
MULTI-CELL APPROACH AND YOU GET
DIFFERENT RESULTS THAN IN THE
SINGLE CELLS?
>> ABSOLUTELY.
WHEN WE COMBINE THE
MULTI-TISSUES INTO INTEGRATED
PLATFORMS WE HAVE A BETTER
UNDERSTANDING OF WHERE THE DRUG
WILL GO SYSTEMICALLY AND WHERE
THE A GOOD EXAMPLE THERE WAS A
LINK OF A KIDNEY AND LIVER CHIP
AND AEROSTOLIC ACID COMMON IN
THE ENVIRONMENT ESPECIALLY WITH
FARMERS THE THE METABOLISM OF
THE DRUG AND WERE ABLE TO
UNDERSTAND HOW IT IMPACTED THE
NEPHROTOXICITY OF THE DRUG WHICH
THEY WOULD NOT HAVE SHOWN ONE
JUST A KIDNEY MODEL.
>> THANKS.
IT LOOKS LIKE A GREAT PROGRAM.
THE TISSUE CHIP INNOVATION AT
NCATS IS WELL KNOWN.
IT'S BEEN GOING ON FOR SOME
TIME.
I LIKE THE FACT YOU HAVE THE
PIPELINE OF PROJECTS AND
INNOVATION AND THE 3-Ds AS
MENTIONED EARLIER AND THEY WILL
NEXT WAVE SEEMS LOGICAL AND FITS
VERY WELL WITHIN THE MAIN GOAL.
ONE OF THE MAIN GOALS OF THE
MISSION WITH MORE TREATMENTS AND
FITS IN LINE WITH GOALS.
NICE JOB.
>> DID YOU HAVE SOMETHING?
ALL RIGHT.
GREAT.
>> TO ADD THERE'S BEEN DIFFERENT
CHIPS THAT EXIST NOT ONLY IN
TERMS OF DEVELOPMENT ACTUALLY
COMMERCIALLY AVAILABLE AT THIS
POINT.
IT'S BEEN GOING ON SINCE 2017.
>> THANK YOU.
KELLY.
ONE QUESTION THAT RELATES AS
WELL AS EARLIER WORK AS SINGLE
TISSUE ON CHIP, HOW TO YOU
ADDRESS THE CONCEPT OF AN
AVERAGE SKIN CELL OR LIVER CELL
AND GROUNDED INTO A PATIENT SO
THE SIMULATION IS -- I'VE BEEN
CURIOUS.
THE SECOND QUESTION IS WHAT
WOULD HAPPEN IF THE STARTING
POINT THERE ARE THE TISSUE OB
CHIP WAS A PLURIPOTENT STEM
CELL.
>> NINE OF THIS TEN TEAMS HAVE
GONE FROM PRIMARY CELLS TO APFCs
TO CREATE MORE PATIENT-MODEL ON
CHIP BUT THAT'S NOT POSSIBLE FOR
SOME CHIPS.
FOR CANCER YOU HAVE TO USE
PRIMARY TISSUE BUT FOR A LOT OF
TISSUE IT'S BEEN ABLE TO GO IN
AND SHOW THE VALUE OF THAT.
THAT'S WHAT THE PROGRAM WILL END
IN ONE MORE YEAR AND WE'LL
FINALLY HAVE DATA.
YOU ASKED SO I'M REPEATING IT
CORRECTLY.
YOU ASKED HOW THE SINGLE TISSUE
CHIPS HOW TO KNOW THE CELL
ITSELF HOW DO YOU KNOW WHICH
CELL TO USE OR --
>> I GUESS I'VE ALWAYS WONDERED
AND -- THIS IS NOT AN AREA OF
EXPERTISE BUT I'VE ALWAYS
WONDERED ABOUT AN AVERAGE CELL
THAT'S EXPERIMENTAL VERSUS A
DISEASE CELL.
IT'S REALLY THE EXPERIMENTAL
CONDITIONS AND IT WAS I THINK MY
SECOND QUESTION WAS REALLY
GETTING TO THE HEART OF THE
FIRST QUESTION I , MAYBE MAYOR
THE FIRST QUESTION.
>> YOU'RE ASKING HOW IS A
TYPICAL CELL, HOW DO YOU KNOW
IT'S DIFFERENT THAN THE DISEASED
CELL?
>> I'LL GIVE A SPECIFIC EXAMPLE.
I DO A GREAT DEAL WITH LUPUS
RESEARCH.
AND WE HAVE MODELS OF TISSUE ON
CHIP THAT LOOK AT KIDNEY ISSUES.
WHEN THEY WERE SHOWING US THIS
WAS A WAY TO START TO DO SOME
MODELLING IN LUPUS, THEY WEREN'T
DOING IT BASED ON CELLS THAT HAD
COME FROM LUPUS PATIENTS.
IT WAS --
>> CELL LINES, BASICALLY.
>> YES.
HOW DOES THIS RELATE AND HOW
DOES THAT IMPACT WHEN YOU'RE
STARTING TO CREATE THESE
MULTI-CELL SYSTEMS?
>> SO, ONE THING WE'VE
ENCOURAGED IS TO NOT USE CELL
LINES I WAS AT A CONFERENCE
WHERE THE CELL LINES WE USE ARE
MALE CAUCASIAN AND DON'T
REPRESENT GENETIC DIVERSITY.
ENCOURAGING THEM TO USE PRIMARY
CELLS FROM PATIENTS OR TO TAKE
CELL LINES AND THEN USE AN AGENT
TO CREATE A DISEASE STATE.
SO USING LPS FOR EXAMPLE TO
CREATE AN INFLAMMATORY STATE AND
SEE IF THAT MIMICS THE NEVER
CONDITION AND THEN TEST AGAINST
THAT.
>> I WANTED THE CLARIFICATION.
IT'S A WONDERFUL PROJECT.
>> JUST TO ADD, THE ADVANTAGE OF
TISSUE CHIPS IS YOU CAN TAKE
PATIENT DERIVED MATERIALS
WHETHER PRIMARY OR BLOOD AND
MAKE THAT INTO PLURIPOTENT STEM
CELLS AND TAKE A CONTROLLED IPS
LINE AND DO SOMATIC CELL GENE
EDITING AND INTRODUCE THE
MUTATION TO HAVE A CONTROL IN
THE BACKGROUND AS PART OF THE
REPLICATES.
YOU CAN ELIMINATE CONTRIBUTIONS
FROM OTHER ALLELES AND CUT DOWN
ON THE MECHANISM OF
PATHOGENESIS.
>> WE'RE GETTING TO THE POINT
WHERE YOU'RE STIMULATING THE
HUMAN AND ELIMINATING THE -- I
MEAN YOU'RE PARALYZING CLINICAL
TRIALS.
>> THAT'S A PLACE WHERE WE'D
LIKE TO SEE HOW CLOSE WE CAN GET
BUT I THINK WE HAVE A LONG WAY.
WE'RE STILL COMPARING THE MODELS
TOO.
HOW CAN WE INCORPORATE IN THIS
NEW CONCEPT OR FOA TO
INCORPORATE THE STANDARDS.
HOW DO WE MAKE SURE WE'RE
COMPARING APPLES TO APPLES WHEN
WE'RE LOOKING AT IPS CELLS FROM
TWO PATIENTS WITH THE SAME
DISEASE?
ARE WE COMPARING APPLES TO
APPLES AND WHAT WOULD BE THE
CHARACTERISTIC OF THAT WHERE WE
WOULDN'T SAY THAT?
IF WE CAN INFUSE THAT INTO THE
DESIGN CONCEPT IS THE STANDARD
OF WHAT IT MEANS TO GET TYPICAL
OR NOT TYPICAL CELL AND ALSO
JUST THINKING ABOUT THE EARLIER
PRESENTATION FROM JEANITA AND
SL
SHARI IS INFUSING THE EQUITY
DESIGN AND STANDARDS IN THESE
MODEL DEVELOPMENTS WILL BE
IMPORTANT AND HOW TO HELP THE
FIELD DERIVE THAT.
THE OTHER FEATURE IS IN THE
BACKGROUND OF ALL THIS IS THE
COMMON FUND PROGRAM
COMPLEMENTARY.
AND HELP DRIVE THE STANDARDS
THERE WILL BE OPPORTUNITIES FOR
INTEGRATING THOSE FINDINGS ALONG
THE WAY.
>> HOPEFULLY THAT ANSWERS YOUR
QUESTION.
>> IT WAS GREAT.
I HAVE FOLLOW-UPS WE'LL TALK
ABOUT OFFLINE WITH RARE DISEASE
COLLABORATIONS.
>> THE MOST WE HAVE IN TERMS OF
PATIENT DIVERSITY IS ABOUT 250
IPS LINES THAT IS BEING USED FOR
METABOLIC ASSOCIATED FATTY LIVER
DISEASE.
IT'S PRIMARILY FROM HISPANIC
POPULATION BECAUSE THEY ARE IN
SOME WAYS THE MOST VULNERABLE
POPULATION FOR THIS PARTICULAR
DISORDER.
AND THEN WE HAVE OTHER DISEASES
AS WELL WHERE LIKE PROSTATE
CANCER UP TO 50 MALE DONORS.
WE ARE INCORPORATING DIVERSITY
BOTH IN TERMS OF ETHNICITY AS
WELL AS SEX AND GENDER.
>> GREAT, THANK YOU.
>> GREAT DISCUSSION.
PAULA.
>> I THINK IT'S A VERY
INTERESTING CONCEPT AND IT HAS
GREAT POTENTIAL.
I KIND OF DOUBT THE TYPICAL CELL
ACTUALLY EXISTS BECAUSE
DEPENDING UPON WHO THE NEAREST
NEIGHBOR IS IT WILL ACT
DIFFERENTLY LIKE KID AND WHOEVER
YOU PUT THEM IN THE ROOM WITH.
THAT'S MAKE A MACRO VIEW AND
YOU'RE LOOKING AT MICRO.
ONE OTHER PART IS IN POTENTIAL
PARTNERS.
SO WE AS CLINICIANS DEAL WITH
THE AVERAGE RESPONSE TO A
CLINICAL TRIAL AND SO YOU HAVE
PEOPLE WHO RESPOND REALLY WELL
AND PEOPLE WHO DON'T RESPOND AT
ALL.
I THINK SOME OF YOUR POTENTIAL
PARTNERS ARE TO TAKE SOME OF
THESE TRIALS WHERE YOU HAVE A
DIVERGENT EITHER A NEGATIVE
RESPONSE OR POSITIVE RESPONSE
AND TRY TO GET THOSE TWO
EXTREMES AND TRY TO USE THAT IN
YOUR SYSTEM TO TRY TO UNDERSTAND
WHY I DIDN'T REACT VERY WELL TO
THIS DRUG AND YET WORKED
BEAUTIFULLY ON SOMEBODY ELSE.
THAT'S A HUGE POTENTIAL PARTNER
SO WE COULD LOOK AT SUBGROUPS AT
TRIALS AND KNOW SOMETHING ABOUT
THE DRUG WORKED FOR YOU OR NOT.
BUT I DO THINK THAT'S ONE OF
YOUR POTENTIAL STAKEHOLDERS IS
LOOKING AT CLINICAL TRIALS AND
TRYING TO FIND THE EXTREMES AND
TESTING THE CHIP ON THAT.
>> THANK YOU.
THAT'S A GREAT SUGGESTION.
I THINK THAT'S ALSO WHY WE LOVE
THE IDEA OF USING MULTI-ORGAN
NPS AND HONE IN ON THOSE AND
ADDRESS OFF TARGET EFFECTS AND
THINGS YOU SEE AND NOT SEE IN
TRADITIONAL IN VITRO STUDIES.
>> ANY OTHER COMMENTS OR
QUESTIONS?
>> OKAY.
IS THERE ANY JUSTIFICATION FOR
YOUR BUILDING THOSE MODELS TO
MIX?
OF AS A WAY TO SCREEN THROUGH
AND THEN DETERMINE THE NOISE?
>> THE DIFFERENT ORGAN SYSTEMS
ARE REALLY MEANT TO RECAPITULATE
NORMAL HUMAN PHYSIOLOGY AND THAT
MEANS MULTIPLE CELL TYPES TO
MAKE SURE WE'RE ACCURATELY
MODELLING.
>> WE HAVE SYSTEMS WHERE PEOPLE
HAVE COMBINED DIFFERENT
PATIENTS.
THAT'S GOING TO BE HARD TO TEASE
ANYTHING OUT OF BECAUSE YOU'RE
GOING TO GET THE NOISE FROM
DIFFERENT PATIENT POPULATIONS.
WE HAVE DEVICES WHERE THEY'VE
DONE THAT AND SOME ARE RESPONSES
WE KNOW.
>> THANK YOU FOR YOUR QUESTIONS.
ANYTHING ELSE?
>> WE NEED TO MOVE ON.
>> IF THERE'S NO FURTHER
DISCUSSION AND I BELIEVE THERE
IS ANY RIGHT NOW CAN I HAVE A
MOTION TO APPROVE THE CONCEPT.
SECOND.
ALL IN FAVOR?
ANY OPPOSED?
ANY ABSTENTIONS?
WITH THAT THE SECOND CONCEPT IS
APPROVED.
THANK YOU VERY MUCH.
>> THANK YOU.
>> OKAY.
AND LAST BUT NOT LEAST, IS OUR
THIRD CONCEPT OF THE DAY THIS IS
COMING FROM THE RARE DISEASE
DIVISION A CONCEPT CLEARANCE PJ
BROOKS WILL BE PRESENTING.
>> THANKS, JONI.
GOOD AFTERNOON EVERYONE.
HAPPY TO PRESENT THE LAST
CONCEPT CLEARANCE OF THE DAY.
ONE WE'RE EXCITED ABOUT.
A LITTLE OVERVIEW.
ONE IS FOCUSSED ON PATIENT
SUPPORT AND INFORMATION AND THE
MAIN RESEARCH INCLUDING A
VARIETY OF PROJECTS FOCUSSED ON
DEVELOPING TREATMENTS FOR A
DISEASE AT A TIME AND HAVE
CONVENING AND PARTNERING
ACTIVITIES.
THE CONCEPTUAL HEAR ABOUT CUTS
ACROSS THE FIRST AND THIRD
PILLARS BUT REINFORCES AN IDEA
THAT GOES THROUGH A LOT OF THAT
MANY DISEASE AT A TIME THINKING.
WITH THAT LET ME HAND IT OVER TO
AINSLIE TISDALE TO PRESENT.
>> SO, I'M BRINGING UP THE REAR
AND THING THIS CONCEPT IS A FUN
WAY TO END THE DAY.
HOPEFULLY YOU'LL AGREE.
I'M ALSO PRESENTING ON BEHALF OF
MY COLLEAGUE JOINING VIRTUALLY
AND WILL BE ABLE TO JOIN IN THE
DISCUSSION SESSION IF WE HAVE
QUESTIONS.
I AM PRESENTING A RENEWAL OF THE
RARE DISEASES ARE NOT RARE
CHALLENGE SOME MAY REMEMBER FROM
YEARS AGO.
IT'S AN EDUCATIONAL AND
AWARENESS TOOL FOR THE RARE
DISEASE SPACE.
SO JUST TO GIVE CONTEXT ON WHY
THIS AWARENESS EDUCATIONAL
CAMPAIGN IS NEEDED AND
IMPORTANT.
THERE'S BEEN A GROUND SWELL IN
THE ATTENTION TO THE COLLECTIVE
BURDEN OF RARE DISEASES
RECENTLY.
THERE'S FOUR STUDIES PUBLISHED
IN 2021 THAT BROUGHT ATTENTION
TO THIS.
AND THE OVER ALL MESSAGE WAS
RARE DISEASE COSTLY ECONOMIC AND
IN HUMAN TERMS.
PATIENTS ARE UNDER RECOGNIZED IN
THE HEALTH CARE SYSTEMS AND
DATABASES AND ACTIONABLE BUT
UNDER PRIORITIZED.
THIS SHOWED RARE DISEASES ARE A
PUBLIC HEALTH PROBLEM THAT
REQUIRE INCREASED ATTENTION,
AWARENESS AND EDUCATION.
NCATS HOSTED THE RARE DISEASES
ARE NOT RARE CHALLENGE IN 2018
AND 2020.
THESE CHALLENGES WERE CENTERED
AROUND RAISING AWARENESS AND
FOSTERING COLLABORATION ACROSS
RARE DISEASE COMMUNITIES
INCLUDING PATIENTS, CAREGIVERS
AND ADVOCATES AND RESULTED IN
SEVERAL COMMUNICATION PIECES
SHARED WIDELY.
SUCH AS POSTERS, VIDEOS, INFO
GRAPHICS, THINGS OF THAT NATURE.
THE SUBMISSIONS OF THIS WINNERS
AND HONORABLE MENTIONS WERE
FEATURED AT OUR RARE DISEASE DAY
NIH EVENT AND THE WINNER WAS
ABLE TO SPEAK AND RECEIVE AN
AWARD.
FOR CONTEXT THE LAST TIME THIS
CONCEPT WAS CLEARED WAS IN
SEPTEMBER OF 2019.
JACOB THOMPSON A RARE DISEASE
PATIENT AND ADVOCATE WAS OUR
FIRST PLACE WINNER IN 2020.
HE PERFORMED SPOKEN WORD POETRY
HE WROTE HIMSELF ABOUT RAISING
AWARENESS ON RARE DISEASED AND
HAD HIM SPEAK AT THE RARE
DISEASE DAY AND HIS VIDEO IS
STILL UP ON THE NCATS WEBSITE
AND YOUTUBE WITH OVER 3,000
VIEWS.
OUR 2018 WINNER WAS A PARENT OF
A CHILD WITH A RARE DISEASE WHO
CREATED FIVE OR SIX OF THESE
POSTERS HERE.
RAISING AWARENESS FOR RARE
DISEASES AND THE PEOPLE THEY
EFFECT DRIVING HOME THE MESSAGE
THAT RARE DISEASES ARE IN FACT
NOT RARE.
WE HAD HARD COPIES OF THE
POSTERS FOR THE 2019 RARE
DISEASE DAY AND THEY'RE STILL
AVAILABLE ON THE WEBSITE AS
WELL.
SO THESE WINNERS WERE
EXCEPTIONAL PIECES BUT AS YOU
SAW WITH OTHER SUBMISSIONS
DURING THE CHALLENGES AND FROM
ANECDOTAL EXPERIENCE AS WELL
PATIENT ADVOCACY GROUPS AND
PARENTS APPROACH RARE DISEASES A
LOT OF THE TIME SINGLE DISEASE
AT A TIME WITH AN UNDERSTANDABLE
APPROACH ON GETTING THEIR
SPECIFIC RARE DISEASE ATTENTION,
TREATMENTS, A CURE, ETCETERA.
AND WE THINK THIS IS
CONSEQUENTLY LED TO A PUBLIC
PERCEPTION THAT RARE DISEASES
ARE INDIVIDUAL PROBLEMS TO BE
ADDRESSED AND RATHER THAN A
COLLECTIVE.
SO WE WOULD LIKE TO THINK ABOUT
THIS IN TERMS OF HOW CANCER HAS
PORTRAYED THEIR MESSAGING AND
CONTRAST HOW THEY MARK CANCERS
AS COLLECTIVE ITEM INSTEAD OF A
DIVERSE SET OF INDIVIDUAL
DISEASES.
CONTRAST THAT WITH THE STARK
TRUTH IN RARE DISEASES.
AS YOU KNOW THERE'S MORE THAN
10,000 RARE DISEASES WITH 5%
HAVING TREATMENTS.
IF WE APPROACH THEM ONE AT A
TIME WE WON'T MEET OUR GOALS OF
GETTING MORE TREATMENTS TO ALL
PEOPLE MORE QUICKLY.
HOW CAN WE CHANGE?
HOW CAN WE CHANGE THE MESSAGING
AROUND RARE DISEASES AS
SOMETHING TO BE APPROACHED
COLLECTIVELY MANY AT A TIME?
TO GIVE HISTORY ON HOW MESSAGING
AROUND RARE DISEASES BEGAN, IT
STARTED TO GAIN TRACTION IN THE
1980s WHEN THE DRUG ACT CAME
INTO EFFECT IN 1983 AND
FOLLOWING THAT UMBRELLA
ORGANIZATIONS WERE FOUNDED.
SOON AFTER NIH ESTABLISHED ORD
NOW DRDRI AND THERE'S BEEN 40
YEARS OF SOME FORM OF MESSAGING
AROUND RARE DISEASE AND RESEARCH
AND THAT MESSAGE HAS NOT BEEN
UNIFIED LUKE OUR CANCER
COUNTERPARTS.
IT'S HAD IMPLICATIONS ON THE
FUNDING RECEIVED FOR RARE
DISEASE RESEARCH.
SO WE PROPOSED THIS CONCEPT AND
ITERATION OF THE CHALLENGE TO DO
JUST THAT.
WE HOPE TO ONE, RAISE AWARENESS
FOR RARE DISEASES AND THE PEOPLE
THEY EFFECT AND HIGHLIGHT THE
NEED FOR CROSS-CUTTING RESEARCH
AND DEVELOPMENT OF NEW TREATMENT
USING MULTIDISEASE MODEL AND
ENGAGEMENT WITH THE RARE DISEASE
COMMUNITY.
SO WHAT'S DIFFERENT WITH THIS
ITERATION OF THE CHALLENGE?
THIS FOCUS OF THE CHALLENGE WILL
BE ON RAISING AWARENESS FOR
DEVELOPING TREATMENT
TECHNOLOGIES FOR MULTIPLE RARE
DISEASES AT A TIME.
AS WE SEE THERE'S A LACK OF
MESSAGING AND PUBLIC
UNDERSTANDING OF THE IMPORTANCE
OF ADDRESSING MANY DISEASES AT A
TIME.
THE GAPS THE CHALLENGE WILL
ADDRESS ARE FIRST INCREASING
EDUCATIONAL OUTREACH TO THE
PUBLIC.
ARTISTIC REPRESENTATIONS CAN
HELP EDUCATE THE LAY PUBLIC AND
HEALTH CARE PROFESSIONALS AND
POLICY MAKE ABOUT THE IMPACT OF
RARE DISEASES ON PATIENTS AND
THEIR FAMILIES AND AMPLIFY THE
PATIENT VOICE PARTICULARLY
WITHIN THE REALM OF RARE
DISEASES AND THE VOICES OF
PATIENTS AND FAMILIES ARE
INVALUABLE.
THE INDIVIDUALS OFFER INSIGHTS
ON LIVED EXPERIENCE CRUCIAL FOR
SHAPING POLICIES AND ADVANCING
RESEARCH.
BY AMPLIFYING THE PATIENT VOICE
THROUGH ACTIVE GOVERNMENT
ENGAGEMENT WE CAN FOSTER AN
ENVIRONMENT NOT ONLY MORE
RESPONSIVE BUT MORE
COMPASSIONATE AND INCLUSIVE.
THE OBJECTIVE TO COMMUNICATE AND
EDUCATE THE PUBLIC THROUGH ART
DISSEMINATED ON VARIOUS
PLATFORMS ABOUT RARE DISEASE.
SLAFT PUB LUCK PERCEPTION IN HOW
WE THINK ABOUT THE RARE DISEASE
PROBLEM COLLECTED INSTEAD OF
INDIVIDUAL AND MAKE RARE DISEASE
DRUG DEVELOPMENT NOR EFFICIENT
BRINGING ATTENTION TO RARE
DISEASES TO GAIN MORE MEDICAL
RESEARCH INTERESTS AND SUPPORT
THERE BE IMPROVING LIVES.
CHALLENGE OFFERS AN OPPORTUNITY
FOR PATIENTS TO BE FUNDED BY
NCATS.
IT'S STILL FUNDING.
THE KEY AREAS OF THE CHALLENGE
ARE THAT THE FOCUS IS ALAINED
WITH OUR NCATS STRATEGIC PLAN WE
SPEND TIME TALKING ABOUT TODAY
AND GOAL 1 OF APPLYING
APPROACHES TO FOSTER THE
IDENTIFICATION, DEVELOPMENT OF
AND ACCESS TO MORE TREATMENTS
AND THE THEME OF DEVELOPING
TREATMENT STRATEGIES TO ADDRESS
MULTIPLE DISEASES AT A TIME.
THE CHALLENGE COULD IMPACT THE
COMMUNITY WITH COLLABORATION AND
HIGHLIGHTING POTENTIAL FUNDING
OPPORTUNITIES, BUILDING A NEW
JERSEY BASE OF HOW THE COMMUNITY
IS APPROACHING THERAPIES FOR
MANY DISEASES AT A TIME AND
ENCOURAGING CROSS-DISCIPLINARY
RESEARCH WITH NOVEL PERSPECTIVES
AND PRO -- APPROACHES WE EXPECT
VARIOUS MEDIA TYPES LIKE SHORT
VIDEOS, POSTERS, INFOGRAPHICS AN
ANIMATED GRAPHICS AS A FEW
EXAMPLES.
IN REGARDS TO THE ONGOING
ACTIVITIES AND HOW THE CHALLENGE
FITS, MANY DISEASES IS A THEME
WITH MANY PROGRAMS AND IT MEANS
TO BROADCAST HOW IT'S PORTRAYED
AND ACCELERATED IN THE
COMMUNITY.
THE NCATS COMMUNICATIONS BRANCH
HAS HAD A CONSISTENT CAMPAIGN TO
EDUCATE THE COMMUNITY ABOUT RARE
DISEASES AND RAISE AWARENESS
GENERALLY.
THE CHALLENGE WOULD FIT IN WITH
THOSE EFFORTS REGARDING TAPPING
IN FOR SUGGESTS AND INPUT.
WHY NCATS?
IT'S SEEN AS THE HOME FOR RARE
DISEASE AT NIH AND AS PART OF
THE CHALLENGE WE COULD RAISE
TRUST GIVING THE COMMUNITY THE
ABILITY TO SHAPE THE RHETORIC
AROUND RARE DISEASES.
DEMONSTRATE THE VALUE OF THEIR
INPUT AND ENGAGEMENT AND AGAIN
THE THEME IS WELL INTEGRATED
WITH OUR STRATEGIC GOAL ONE THE
THEME OF DEVELOPING TREATMENT
STRATEGIES FOR ADDRESSING
DISEASES AT A TIME AND THE THEME
FOR ALL PEOPLE WHO HAVE A
VARIETY OF DISEASES.
THE IMPACT ON THE RESEARCH
COMMUNITY.
WE SEE THE CHALLENGE FOSTERING
INNOVATION AND COLLABORATION AND
INCREASE AWARENESS AND HIGHLIGHT
POTENTIAL FUNDING OPPORTUNITIES,
ACT AS A TOUCH POINT TO BUILD
THE KNOWLEDGE BASE HOW THE
ACOMMUNITY IS APPROACHING MANY
DISEASES AND RAISE AWARENESS
ABOUT NCATS' ROLE IN THE RARE
DISEASE COMMUNITY.
SUCCESS.
WE SEE SUCCESS LOOKING AT
GREATER THAN 30 SUBMISSIONS
WHICH IS MORE THAN WE HAVE IN
THE PAST.
WE'D LIKE TO SEE INCREASED
VISITS TO THE NCATS RARE DISEASE
WEB PANEL TO REFLECT THE
INCREASED AWARENESS AND USE THE
SUBMISSIONS AS CREATIVE
MECHANISMS WORKING TOGETHER AND
PUSH THE NARRATIVES THAT RARE
DISEASE CAN BE ADDRESSED MANY AT
A TIME AND HAVE WINNERS AND
HONORABLE MENTIONS SHARED ON
SOCIAL MEDIA AS PART OF THE
AWARENESS CAMPAIGN AND
ULTIMATELY HAVE INCREASED
COLLABORATION AND NETWORKING.
SO, SO I'LL END WITH A FEW OVER
ARCHING AREAS OF IMPACT.
WE WANT TO HIGHLIGHT NCATS ROLE
IN THE RARE DISEASE COMMUNITY AS
STATED WITH THE HOME FOR RARE
DISEASES AT NIH.
AS A GOVERNMENT RESEARCH AGENCY
WE WANT TO PUT THE MESSAGE OUT
HOW IMPORTANT THE PATIENT VOICE
IS AND AMPLIFY THE PATIENT VOICE
AND INCREASE AWARENESS AND
VISIBILITY OF RARE DISEASE TO
GAIN MORE INTEREST AND SUPPORT
THERE BE IMPROVING LIVES.
IN THE RARE DISEASE SPACE
COMMUNITY IS THE DRIVER OF
RESEARCH.
WE SEE THEM AS THE ONCE WHO
APPROACH SCIENTISTS AND SAY HEY,
MY DAUGHTER HAS THIS DISEASE AND
I'VE DONE THIS AMOUNT OF
RESEARCH AND THINK YOU'RE LAB
SPECIFICALLY CAN HELP ME.
THIS CHALLENGE IS SUCH A
MEANINGFUL WAY TO COLLABORATE
WITH THE RARE DISEASE COMMUNITY
AND CREATE A MESSAGING CAMPAIGN
TOGETHER WHICH IS WHAT I'M MOST
EXCITED ABOUT WITH THIS
CHALLENGE AND ULTIMATE CHANGE
THE PUBLIC PERCEPTION OF RARE
DISEASES BRINGING MORE TRE
TREATMENTS TO MORE PEOPLE MORE
QUICKLY.
I'LL END WITH OUR DISCUSSION
SLIDE.
ALONG WITH THE GENERAL QUESTIONS
WE HAVE FOR COUNCIL WE HAVE TWO
SPECIFIC QUESTION.
THE FIRST BEING IF THERE'S ANY
SUGGESTIONS FOR PATIENT ADVOCACY
GROUPS OR ORGANIZATIONS NOTIFY
TO COORDINATE SUBMISSION AND IF
YOU HAVE SUGGESTIONS HOW TO SET
UP THE CHALLENGE WEBSITE WITH
THE DESCRIPTION TO GET RESPONSES
THAT GET AT THE COLLECTIVE
APPROACH INSTEAD OF THE PREVIOUS
RESPONSES WE GOT BEFORE THAT
WERE MORE TARGETED ON INDIVIDUAL
APPROACH.
AND OF COURSE ANY OTHER
COMMENTS.
I'LL END THERE.
>> THANK YOU.
STARTING WITH AMY.
>> YOU PROBABLY WON'T BE
SURPRISED BUT I LOVE THIS.
IN ALL SINCERITY I THINK FULL
DISCLOSURE I WAS PART OF TWO AND
THE FACT THAT AN PUBLIC AGENCY
WANT TO DO A CAMPAIGN ABOUT THE
FACT THAT RARE DISEASE IS A
PUBLIC HEALTH CRISIS AND THAT IN
AND OF ITSELF IS AMAZING.
THAT SAID I THINK THE FACT THAT
THE NATIONAL ECONOMIC BURDEN OF
RARE DISEASE STUDY WAS CONDUCT
AND INFORMED BY THE RARE DISEASE
COMMUNITY.
FOR THOSE WHO MAY NOT BE AWARE
THE FIRST STUDY ON THE SLIDE
INCLUDED DIRECT MEDICAL COST
DATA AND INDIRECT AND
NON-MEDICAL DATA.
TO COLLECT THAT DATA INCLUDED A
SURVEY CREATED BY BIG COMMUNITY.
WE WORKED TOGETHER TO DEVELOP
THE SURVEY AND DATA ELEMENTS WE
WOULD DISSEMINATE THROUGHOUT THE
COMMUNITY.
AND DISSEMINATE IT TO THOSE
REGISTERED WITHIN THEIR
MEMBERSHIP.
THE TAGS DISSEMINATED IT AND
COLLECTED DATA TO WORK TO DO THE
DATA ANALYSIS AND USED THE
DATABASES.
THAT'S HOW THE STUDY WAS DONE.
AS WE DISSEMINATED THE FINDINGS
WE WORKED WITH MANY LEADERS TO
DO THE DISSEMINATION.
THE RARE DISEASE COMMUNITY HAS
BECOME VERY ADEPT AT
COMMUNICATING ABOUT THE IMPACT
OF THE COLLECTIVE ECONOMIC
BURDEN OF RARE DISEASE.
AND ALSO LEARNED BURDEN DOESN'T
MEAN IT'S A BURDEN TO HAVE A
LOVED ONE WITH RARE DISEASE BUT
FINANCIAL BURDEN.
IT'S A VERY DIFFERENT CLIMATE TO
THINK ABOUT PUTTING THIS OUT NOW
THAN IT WAS WHEN YOU DID IT THE
LAST TIME.
THEN THAT PAIRED WITH THE STUDY
NCATS LED AND PUBLISHED AND
OTHER OP-EDS.
WE'VE CHANGE THE WAY WE TALK
ABOUT RARE DISEASE IN THE U.S.
THAT'S THE FIRST THING.
THE OTHER THING IS AS FAR AS
PARTNERING MANY TAGS AS PART OF
THE WORK, US INCLUDED, WHO WOULD
BE EXCITED HELPING DISSEMINATE
AND PUSH THIS OUT.
I WOULD ALSO FLAG WE HELP
COORDINATE THE RARE DISEASE
CAUCUS AND ALSO LEAD A RARE
ARTIST CAMPAIGN EVERY YEAR AND
DURING RARE DISEASE WEEK MARRIED
TO YOUR RARE DISEASE DAY WE HAVE
A RARE ARTIST RECEPTION ON
CAPITOL HILL ATTENDED BY CAUCUS
MEMBERS AND THAT MAY BE A NICE
OPPORTUNITY TO HAVE WHOEVER THE
WINNER OF THE CHALLENGE TO
FEATURE THEIR ART.
THERE MAY BE NICE SYNERGIES AND
TALKING ABOUT RAISING THE SCO ON
YOUR WEB PAGES HAVING THEM
INCLUDED ON OUR WEB PAGES IS
ANOTHER PARTNER TO INCLUDE HERE.
THEY'RE INTERESTED IN THESE
PUBLIC HEALTH CAMPAIGNS AND
MAKING SURE PEOPLE KNOW RARE IS
NOT RARE.
THERE'S NATURAL PARTNERS TO
THINK OF.
AMAZING PROGRAM.
>> THANKS, ANNIE.
>> THANK YOU.
KELLY.
>> I'M SO EXCITED YOU'RE
AMPLIFYING THE VOICE OF THE
PATIENT IN THIS WAY AND HAVE A
TECHNICAL QUESTION.
WOULD YOU CONSIDER ALLOWING THE
SUBMISSIONS IN EN ALL THE MEDIA
THAT COULD HAVE A DIGITAL
COMPONENT TO BE MINTED RATHER
THAN SUBMITTED TO BECOME A
NON-FUNGIBLE TOKEN TO PRESERVE
THE PROPERTY RIGHTS OF THE
PEOPLE GENERATING THE ART?
>> GOOD QUESTION.
THE PAST TWO CHALLENGES WE HAD
SPECIFIC LANGUAGE THAT SAID WE
NOW OWN IT SO I DON'T KNOW HOW
WE'D GO ABOUT DOING IT FOR THIS
UPCOMING CHALLENGE.
>> ANNA IS OUR
CHIEF-LOOKER-INTO.
GREAT IDEA.
GREAT QUESTION.
WE'LL GET BACK TO YOU.
>> THANK YOU.
HAVE OUR FINAL DISCUSSANT IS
ROBIN.
>> THANKS.
I SUPPORT THIS LOVELY CONCEPT
AND IT'S NICE TO END THE DAY ON
AN UPBEAT CONCEPT.
I'LL PUSH YOU A LITTLE BIT GIVEN
IT'S YOUR THIRD ROUND OF DOING
THIS AND HELP YOU THINK THROUGH
HOW TO EVALUATE IT IN TERMS OF
REACHING DIVERSITY BECAUSE YOU
DON'T WANT THE SAME PEOPLE
SUBMITTING AND HAVE YOU
BROADENED YOUR REACH AND WHO'S
INCLUDED AND EXCLUDED AND
LOOKING AT THAT FROM MULTIPLE
DIMENSIONS WHAT ARE DISEASES
THAT ALWAYS COME UP AND
OVERREPRESENTED.
WHO WITHIN THE DISEASE GROUPS
YOU WANT IT TALK ABOUT
MINORITIZED COMMUNITIES HAVING
FINANCIAL TOXICITY FOR RARE
DISEASE AS EXCESSIVE.
MAYBE MAKING AN EFFORT TO
BROWEDEN THE REACH THIS TIME TO
GET -- BROADEN THE REACH TO GET
THE UNSEEN ONES AND ELEVATE
THEM.
>> YEAH.
I APPRECIATE THAT SUGGESTION.
IT'S SOMETHING WE WILL CONSIDER
WHEN WE'RE PLANNING THE
PROMOTION AND DISSEMINATION OF
THE CHALLENGE.
I THINK WE ALSO BROUGHT UP
INTERNATIONAL EFFORTS AS WELL.
PJ'S A PART OF.
WE HAD CONVERSATION ABOUT RARE
DISEASE DISEASE AND EXPANDING
THE REACH.
I THINK SUGGESTIONS MADE IN THE
CONVERSATION COULD PLAY IN THE
CHALLENGE AS WELL.
>> WITH THE INTERNATIONAL
APPROACHES WE CAN EXPAND TO
INTERNATIONAL GROUPS BUT THEY
WILL NOT BE ELIGIBLE TO RECEIVE
A PRIZE BUT WOULD STILL BE
ELIGIBLE TO SUBMIT.
>> AND NOT TO RECEIVE THE
FINANCIAL PRIZE BUT IF THEY WERE
TO WIN, WE COULD TRAVEL THEM TO
SPEAK AT RARE DISEASE DAY.
>> THERE'S AN INTERNATIONAL
BURDEN OF RARE DISEASE STUDY
ABOUT TO BE SUBMITTED FOR
PUBLICATION.
WE'VE BEEN PARTNERING WITH
OTHERS ON THAT STUDY.
>> WE'D LOVE AN ON TIME EARLY
COPY.
>> WE WOULD.
>> OR SAME TIME.
OKAY.
I TOOK OVER.
YOU'RE DOING GREAT.
WE'RE DONE.
>> ALL RIGHT.
IF THERE'S NO ADDITIONAL
DISCUSSION CAN I HAVE A MOTION
TO APPROVE THE LAST CONCEPT OF
THE DAY.
SECOND?
ALL IN FAVOR?
ANY ABSTENTIONS?
WITH THAT THE LAST CONCEPT OF
THE DAY IS APPROVED.
THANK YOU VERY MUCH.
>> OKAY.
I THINK THAT DOES IT FOR THE DAY
EXCEPT FOR ANOTHER HOUR OF
PRESENTATION -- JUST KIDDING.
I WANTED TO JUST REMIND YOU TO
FILL OUT YOUR FORMS.
MAKE THE COUNCIL TEAM HAS THEM.
PLEASE ALSO GIVE YOUR BADGES
BACK TO THE FRONT DESK.
WE RECYCLE THOSE.
AND ALSO I WANT TO TAKE A MOMENT
TO THANK AGAIN THE COUNCIL TEAM
FOR PREPARING FOR TODAY.
I KNOW THIS IS ALWAYS A BIG
MEETING TO PREPARE FOR.
JUST THANK YOU AGAIN FOR THE
COUNCIL'S WORK.
THANK YOU FOR THE GREAT CONCEPT
DISCUSSIONS.
THIS IS REALLY WHAT'S SO
IMPORTANT AND WHAT MAKES NCATS
SO SUCCESSFUL.
I APPRECIATE YOUR PARTICIPATION
TODAY.
ALSO, TWO MORE THINGS.
ONE IS I WANT TO THANK OUR
COUNCIL FOR YOUR TREMENDOUS
LEADERSHIP AND SEEING WHAT'S
POSSIBLE.
AND HELPING US TO DRIVE NCATS I
THINK MORE THAN WHERE WE THINK
WE CAN EVEN GO.
THE LETTER YOU SENT US THIS WEEK
WAS A BIG WAY OF SHOWING THIS.
AND I FEEL VERY FORTUNATE TO
HAVE SUCH A DYNAMIC COUNCIL
MEMBERS AND TEAM WHO REALLY
BRINGS IT EVERY TIME.
SO THANK YOU FOR DOING THAT.
AND ANNIE, JUST ANOTHER REMINDER
OR PERHAPS THE REMINDER IS
YOU'RE NOT GOING TO GO FAR.
WE KNOW WHERE YOU LIVE.
BUT WE'LL MISS YOU AND THOUGH
YOU WON'T BE A MEMBER ANY
LONGER, WE KNOW AGAIN HOW TO
CONTINUE TO WORK WITH YOU IN THE
SPACES WE CARE ABOUT MUTUALLY.
THANK YOU FOR YOUR EFFORTS ON
THE COUNCIL AND WE WERE YOU THE
BEST IN YOUR NEXT ENDEAVORS.
THANK YOU.
WITH THAT I'LL GAVEL US OUT.
OH, PUBLIC COMMENTS.
YES, WE HAVE TIME.
ARE THERE ANY PUBLIC COMMENTS?
>> WOULD ANY MEMBER OF THE
PUBLIC LIKE TO MAKE A COMMENT?
ALL RIGHT.
>> IN THAT CASE, I'LL GAVEL US
OUT.
THANK YOU ALL.
SEE YOU NEXT TIME.