THANK YOU SO MUCH FOR
PARTICIPATING IN OUR 35th
MEETING OF THE NATIONAL CENTER
FOR ADVANCING TRANSLATIONAL
SCIENCES, OUR ADVISORY COUNCIL
TODAY.
IT'S A WINTER WONDERLAND HERE IN
MARYLAND.
AND WE HAVE MOST OF OUR STAFF
WORKING FROM HOME AND SOME OF US
ARE HERE.
I WANT TO THANK THE COUNCIL
MEMBERS WHO HAVE ALSO BEEN
EXPERIENCING WEATHER AS WELL AND
APPRECIATE YOUR ATTENDANCE TO
THE MEETING TOO.
HOPEFULLY THINGS WILL GO RATHER
SMOOTHLY TODAY.
THIS IS WHY WE DO OUR MEETINGS
VIRTUALLY IN JANUARY SO WE CAN
ACTUALLY ACCOMMODATE THIS SORT
OF THING, SO I'M HOPING IT WILL
GO ALONG WELL.
IF YOU HAVE ANY TROUBLE ALONG
THE WAY PLEASE DO LET US KNOW,
AND WE'LL TRY TO HELP YOU OUT
ALONG THE WAY.
I WANTED TO MAKE A QUICK
ANNOUNCEMENT.
MANY OF YOU KNOW ANNA
RAMSEY-EWING, SHE'S REALLY THE
PERSON WHO RUNS THESE COUNCILS
AS OUR EXECUTIVE OFFICERS FOR
THE COUNCIL MEETINGS, OUR
DIRECTOR OF THE DIVISION OF
EXTRAMURAL AFFAIRS AT NCATS.
SHE'S FEELING A LITTLE BIT UNDER
THE WEATHER, AND SHE'S FEELING
FINE BUT FEELS LIKE SHE MAY NOT
BE ABLE TO GET THROUGH SOME
TALKING SO SHE HAS ASKED HER
DEPUTY TO ALSO SORT OF MANAGE
AND MODERATE THE MEETING, SO HE
WILL TAKE THE REINS.
ANNA WILL BE ON FOLLOWING
THROUGH BUT WANTED TO GIVE HER A
BREAK AND I'M SURE GREG DID TOO
AND MAKE SURE SHE'S GOING TO
FEEL WELL AS WE GO FORWARD.
ANNA, WE'RE HERE FOR YOU, AND
WE'VE GOT YOUR BACK FOR THIS.
GREG, I WILL TURN IT OVER TO YOU
TO TAKE THE ROLL CALL AFTER I
GAVEL US IN.
WELCOME TO THE MEETING,
EVERYONE.
GREG, OVER TO YOU.
>> OKAY.
THANK YOU, JONI.
NEXT SLIDE.
WE'LL START WITH THE ROLL CALL
BEFORE WE GO INTO THE AGENDA.
FIRST UP, SERGIO GAXIOLA.
>> HERE; IT'S AGUILAR.
>> THANK YOU.
PAUL HARRIS.
>> HERE.
>> GREAT.
MATIAS PRESS LEER.
>> YES.
>> KELLY McVEERY.
>> ROBIN NORMALSTEIN.
>> HERE.
>> KEITH MULLER.
>> HERE.
>> PAULA SHERMAN.
>> HERE.
>> MARSHAL SUMMER.
>> HERE.
>> ANIKA WAYMAN.
>> HERE.
>> GREAT.
WE DO NOT HAVE ANY COUNCIL
NOMINEES ATTENDING AS AD HOCS,
ANNIE KENNEDY IS NOT PRESENT.
LET'S GO OVER THE AGENDA WITH
SOME ANNOUNCEMENTS AND REMINDERS
FOLLOWED BY JONI PROVIDING THE
NCATS DIRECTOR'S REPORT, THEN
SPECIAL PRESENTATION BY OUR
GUEST SPEAKER DR. MONICA
BERTAGNOLLI, DIRECTOR OF NIH, A
SHORT 15-MINUTE BREAK.
AFTER THE BREAK TWO PROGRAM
UPDATES, FIRST FROM OFFICE OF
SPECIAL INITIATIVES, PRESENTED
BY DAN TAGLE AND HIS TEAM, AND
AFTER THAT THE OFFICE OF
STRATEGIC ALLIANCES WILL PRESENT
THEIR PROGRAM UPDATE PRESENTED
BY KRISHNA BALAKRISHNAN AND HIS
TEAM.
PLEASE USE THE "RAISE HAND"
FEATURE AND WAIT UNTIL CALLED ON
TO PROVIDE INPUT.
THIS IS A PUBLIC MEETING.
ANYTHING IN THE CHAT BECOMES
PART OF THE PUBLIC RECORD,
INCORPORATED INTO THE MINUTES.
A TRANSCRIPT OF THE CHAT IS KEPT
FOR THE RECORD.
SO PLEASE KEEP THE USE OF THE
CHAT TO A MINIMUM.
PLEASE ALSO ALLOW SPEAKERS TO
MOVE THROUGH PRESENTATIONS,
NOTING YOUR COMMENTS AND
PRESENTING THEM WHEN CALLED
UPON.
WE'RE USING THE VIDEOCAST
FEATURE SO AUDIENCE MEMBERS MAY
SUBMIT COMMENTS UNTIL FEBRUARY 2
BY USING THE "SEND LIVE
FEEDBACK" BUTTON OR BY SENDING
AN E-MAIL TO THE E-MAIL ADDRESS
SHOWN.
PLEASE BE SURE WHEN YOU ARE
SPEAKING YOUR CAMERA IS ON SO
YOU ARE SPOTLIGHTED FOR VIEWERS
OF THE VIDEOCAST.
IF NOT ON CAMERA THE PREVIOUS
SPEAKER REMAINS SPOTLIGHTED.
NEXT SLIDE PLEASE.
HERE'S A LIST OF THE FUTURE
NCATS ADVISORY COUNCIL MEETINGS
IN 2024, MAY 23 AND SEPTEMBER 19
ARE IN-PERSON MEETINGS, IN 2025
JANUARY MEETING IS VIRTUAL
JANUARY 30-31, TWO IN-PERSON
MEETINGS ON MAY 22 AND SEPTEMBER
18.
NEXT SLIDE PLEASE.
IN JANUARY WE'RE SEEKING
APPROVAL OF NCATS ADVISORY
COUNCIL 2024 GENERAL OPERATING
PROCEDURES, PROVIDED AHEAD OF
TIME FOR YOUR REVIEW AND ARE
AVAILABLE IN THE ECB, ELECTRONIC
COUNCIL BOOK.
SO COULD WE HAVE A MOTION TO
APPROVE THE 2024 GENERAL
OPERATING PROCEDURES.
>> SO MOVED.
>> SECOND.
>> IN FAVOR.
>> AYE.
>> ANY OPPOSED, ANY ABSTENTIONS
IN GREAT.
THE 2024 GENERAL COUNCIL
OPERATING PROCEDURES ARE
APPROVED.
NOW I'LL TURN IT BACK TO JONI
FOR THE DIRECTOR'S REPORT.
>> .
>> THANKS, GREG.
I HAVE A VARIETY OF THINGS TO
RELATE AND I WILL JUMP RIGHT IN.
AS I HAVE BEEN DOING IN THE
PAST, I PULL OUT THE SUMMARY
FROM MY PREVIOUS COUNCIL
PRESENTATION TO REMIND ME AND
ALL OF YOU WHAT WAS PRESENTED
LAST TIME, AND SO AT THE LAST
MEETING PRESIDENT BIDEN HAD
NOMINATED DR. BERTAGNOLLI AS NIH
DIRECTOR, AND I'LL TALK MORE
ABOUT THAT.
WE'RE REALLY LUCKY TO HAVE HER
HERE TODAY TO SPEAK AFTER MY
PRESENTATION.
I HAD ASKED HER ACTUALLY TO
PRESENT WHEN SHE WAS NCI
DIRECTOR, AND KIND OF JUST
WORKED OUT, SO REALLY HAPPY TO
HAVE HER HERE LATER TODAY.
I ALSO TALKED ABOUT HOW NCATS
LEADERSHIP IS GROWING, I'LL TALK
MORE ABOUT THAT AGAIN BECAUSE IT
CONTINUES.
AND A VARIETY OF IMPACTFUL
ADVANCES THAT I'LL PICK UP WHERE
WE LEFT OFF LAST TIME.
SO, THAT'S WHERE WE WERE.
HERE'S SOME OF THESE ACTIVITIES
THAT HAVE HAPPENED TO DATE.
MONICA BERTAGNOLLI IS NOW THE
NIH DIRECTOR, SHE'S HAD A COUPLE
INTERVIEWS.
SO I'VE HIGHLIGHTED THE ONE SHE
DID IN THE NIH RECORD HERE TO
TALK ABOUT HER PRIORITIES,
LARGELY RELATED TO ENSURING
CLINICAL TRIALS YIELD THE BEST
RESULTS, INCREASING DIVERSITY OF
PARTICIPANTS IN CLINICAL
RESEARCH, USING LEARNING-BASED
ANALYTICAL TOOLS, AND ENSURING
THEIR USE IMPROVES CARE FOR ALL,
RESTORING TRUST IN SCIENCE,
INSPIRING THE NEXT GENERATION OF
DOCTORS AND SCIENTISTS,
LEVERAGING COMMONALITIES ACROSS
DISEASES.
I PULLED OUT HIGHLIGHTS, NEAR
AND DEAR TO OUR HEARTS AS WELL
AT NCATS.
THERE'S A LOT OF ALIGNMENT IN
THE VISION AND I'M EXCITED TO
HELP THIS TAKE SHAPE.
AND AS I MENTIONED, SHE WILL BE
HERE LATER TODAY AT 1:45 TO GIVE
HER OWN VISION AND PRIORITIES
AND TALK TO YOU MORE ABOUT
THOSE.
SO WE'RE EXCITED FOR THAT.
IN ADDITION TO SOME OTHER
LEADERSHIP ACTIVITIES, THE NEW
NCI DIRECTOR, AS DR. BERTAGNOLLI
LEFT, IT WAS PRETTY QUICK TURN
AROUND IN TERMS GESTING THE NEXT
NCI DIRECTOR, DR. KIMRYM
RATHMELL, ANOTHER REAL STAR HERE
AT NCI TO LEAD THAT INSTITUTE.
WE HAVE AN OPENING AT NATIONAL
LIBRARY OF MEDICINE TO REPLACE
DR. PATTI BRENNAN AND FOGARTY
INTERNATIONAL CENTER.
BOTH THESE POSITIONS ARE IN
VARIOUS STAGES OF THEIR
ACTIVITIES, SO WE'RE HOPING TO
HEAR SOON ON ONE, BUT THE THINK
THE NLM IS FURTHER BEHIND.
NOW, IN ADDITION TO SOME COMINGS
AND GOINGS, AS YOU KNOW THERE'S
A TERM LIMIT ON YOUR
PARTICIPATION IN COUNCIL.
THIS TIME WE'RE -- FOR THIS
COUNCIL WE'RE SAYING FAREWELL TO
TWO COUNCIL MEMBERS, THESE
THINGS AREN'T EASY WHEN WE HAVE
GROWN ACCUSTOMED TO ONE ANOTHER
BUT I WANT TO RECOGNIZE DR.
MATTIAS KRETZLER, A FANTASTIC
MEMBER ON COUNCIL FROM THE
UNIVERSITY OF MICHIGAN AT ANN
ARBOR, HAS WORKED IN THE RDCRN
PROGRAM AND JUST GENERAL
EXCITEMENT AROUND TRANSLATIONAL
SCIENCE.
THEN THE OTHER COUNCIL MEMBER
WHO IS LEAVING US IS DR.
MARSHALL SUMMER, NOW CHIEF
OFFICER IN WASHINGTON, D.C. AND
HAS BEEN SUCH A CHAMPION OF THE
RARE DISEASE WORK SAD TO SEE
THEM GO, IT'S ALWAYS DIFFICULT
TO SAY FAREWELL.
DURING THEIR TENURE, THEY HAVE
NOT JUST BEEN SORT OF THE
ADVISORS THAT YOU ALL ARE, BUT
ALSO THE BEST CRITICS AND
DEEPEST ALLIES THAT WE CAN HAVE.
THAT'S SUPER IMPORTANT FOR WHAT
WE'RE TRYING TO DO.
THE WORK WE DO TOGETHER IS
REALLY MADE BETTER BY OUR
CONVERSATIONS.
SO YOUR INSIGHTS, DEDICATION AND
UNWAVERING SUPPORT HAVE BEEN
INSTRUMENTAL IN GUIDING OUR
INITIATIVES AND DECISIONS
ESPECIALLY IN THE SPACE OF RARE
DISEASES AND THERAPEUTIC
DEVELOPMENT, AND NEW PREDICTIVE
MODELS.
SO MATTIAS AND MARSHALL, YOUR
CONTRIBUTIONS SHAPED OUR WORK,
THIS IS NOT A GOOD-BYE BUT MORE
OF A "WE'LL BE IN TOUCH SOON"
AND I WANT TO EXTEND MY
GRATITUDE FOR YOUR SERVICE AND
DEDICATION.
THANK YOU FOR BEING INTEGRAL
MEMBERS OF OUR NCATS TEAM.
OUR VIRTUAL ENVIRONMENT MAKES
THIS LESS TANGIBLE BUT PLEASE, A
ROUND OF APPLAUSE FOR THE TWO
MEMBERS AND THE SERVICE.
SO THANK YOU.
>> JONI, IT'S BEEN TREMENDOUS
FUN TO DO THIS.
REALLY APPRECIATE, DEEPLY
APPRECIATE, THE OPPORTUNITY TO
DO THIS.
I'VE LEARNED SO MUCH.
I LEAVE KNOWING THE PLACE IS IN
GREAT HANDS UNDER YOUR
LEADERSHIP, AND SO EXCITED TO
HAVE NEW LEADERSHIP WITH THE
RARE DISEASE GROUP WITH
DOMINIQUE.
SO JUST SUCH A GOOD JOB YOU'RE
DOING.
>> THANK YOU.
THANK YOU, MARSHALL.
>> CONGRATULATIONS FOR AN
OUTSTANDING TRANSITION AND
EXCITING TIME FOR NCATS.
YES, PLEASE CALL UP ON US WHEN
WE CAN BE IN ANY WAY, SHAPE, OR
FORM HELPING YOU ON WHAT HAS
BEEN AN OUTSTANDING START.
THANKS SO MUCH.
>> FANTASTIC.
>> ABSOLUTELY.
>> THANK YOU.
THANK YOU FOR THAT.
YES, OUR VIRTUAL ENVIRONMENT
MAKES THIS LESS TANGIBLE BUT IT
IS NO LESS FELT.
THANK YOU FOR THE ROUND OF
APPLAUSE TOO.
ON A SAD NOTE WE'VE LEARNED THAT
AN NCATS COUNCIL MEMBER HAS
PASSED AWAY LAST YEAR.
I MET RAJESH MANY YEARS AGO WHEN
HEADING TRANSLATIONAL EFFORTS AT
NINDS, AND HE HELPED LAUNCH THE
BLUEPRINT NEUROTHERAPEUTICS
PROGRAM OUT OF THE -- SOME
INSTITUTES AND CENTERS ACROSS
THE NIH, AND HE WAS ALSO PART OF
A CORE GROUP AT THE NIH THAT WAS
INSTRUMENTAL IN SUPPORTING THIS
NEW I.C. FOCUSED ON
TRANSLATIONAL SCIENCE NOW KNOWN
AS NCATS.
HE PLAYED A PIVOTAL ROLE AND
INITIATED TRANSLATIONAL SCIENCE
PROGRAM OUT OF THE OFFICE OF
INTRAMURAL TRAINING AND
EDUCATION, AND HE AND I SHARED
THE HONOR OF KICKING OFF THAT
TWO-DAY IMMERSIVE TRAINING
PROGRAM.
HE REALLY HAD THIS PASSION FOR
TRAINING AND HE HAD THIS ALSO
NO-NONSENSE WAY OF DELIVERY THAT
I REALLY GREW TO APPRECIATE.
AND HE JOINED THE NCATS COUNCIL
WHEN I WAS ACTING DIRECTOR, AND
AM TRULY SADDENED BY HIS LOSS
AND WANTED TO SHARE THAT WITH
YOU AS WELL.
IN TERMS OF THE NCATS
LEADERSHIP, THERE HAVE BEEN A
VARIETY OF DIFFERENT TRANSITIONS
AS YOU'VE HEARD ABOUT AT OUR
LAST MEETING.
I WANTED TO LET YOU KNOW STATUS
OF WHERE WE ARE NOW.
WE ARE VERY CLOSE TO ANNOUNCING
OUR SCIENTIFIC DIRECTOR AND OUR
OFFICE OF TRANSLATIONAL MEDICINE
POSITIONS.
WE HAVE -- WE'RE SORT OF IN THE
MIDST OF THE PROCESS FOR THE
DEPUTY DIRECTOR POSITION.
AND THE CHIEF OF STAFF POSITION.
SO I HOPE THAT BY OUR NEXT
MEETING YOU WILL HEAR THE FULLY
FORMED LEADERSHIP AT NCATS.
SO, IT'S TAKEN US A WHILE TO GET
HERE BUT I FEEL LIKE WE'RE
REALLY IN VERY GOOD SHAPE IN
THAT REGARD.
SO, THAT SAID, IT IS ALSO THE
LAST COUNCIL FOR OUR DEPUTY
DIRECTOR, CLARE SCHMITT.
WE'VE GONE THROUGH THE PROCESS
OF SEARCHING FOR A NEW DEPUTY,
SO I WANTED TO SAY A THANK YOU
TO CLARE FOR THREE YEARS, HAS
SERVED AS ACTING DEPUTY
DIRECTOR, GRACIOUSLY EXTENDING
HER COMMITMENT FOR ANOTHER YEAR
TO THIS YEAR.
SHE WAS HOPING TO RETIRE LAST
YEAR BUT EXTENDED THAT A LITTLE
BIT.
AND HER PARTNERSHIP, LEADERSHIP,
HAVE BEEN INVALUABLE.
SHE HAS THIS MASTERY OF
EMOTIONAL INTELLIGENCE THAT HAS
BEEN INCREDIBLY VALUABLE TO ME
AND A GUIDING LIGHT FOR A LOT OF
US HERE.
HER JOURNEY WITH NIH BEGAN IN
2000, SINCE THEN SHE'S BEEN
WORKING IN THE FIELD OF
MICROBIOLOGY WITH VERY KEEN
FOCUS I THINK ON MICROBIAL
PATHOGENESIS, FOOD AND
WATER-BORNE ILLNESSES,
TRANSLATION OF BASIC SCIENCE
INTO LIFE SAVING SOLUTIONS,
DURING THE COURSE OF THE
PANDEMIC SHE WAS INSTRUMENTAL IN
HELPING US NAVIGATE THAT.
AS SHE PREPARES TO EMBARK ON
WELL-DESERVED ENVIRONMENT I FEEL
PROFOUND GRATITUDE FOR HER
WISDOM AND THE WARMTH SHE
BROUGHT, THIS SENTIMENT IS
SHARED ACROSS NCATS.
CLARE, YOU'VE BEEN MORE THAN A
DEPUTY DIRECTOR.
YOU'VE BEEN A MENTOR, LEADER,
AND A FRIEND.
ON BEHALF OF EVERYONE HERE I
THANK YOU SO MUCH FOR YOUR
EXTRAORDINARY SERVICE AND I WISH
YOU ALL THE BEST IN YOUR
RETIREMENT.
SO THANK YOU, CLARE.
AGAIN, NOT TANGIBLE BUT VERY
HEARTFELT, SO THANK YOU.
AND THEN ON TO SORT OF THE
MATTERS MORE BUSINESS-LIKE
MATTERS, FILLING YOU IN ON THE
APPROPRIATIONS NEWS AND BUDGET
ACTIVITIES THAT ARE ONGOING.
AT OUR LAST COUNCIL I MENTIONED
WE HAD NOT HAD CLARITY ON THE
BUDGET AS YET.
THAT REMAINS TO BE THE CASE AS
WE STAND NOW.
BUT THERE HAS BEEN RECENT
ACTIVITY.
NCATS IS OPERATING UNDER A
CONTINUING RESOLUTION THROUGH
MARCH 8 THAT WAS JUST DONE
YESTERDAY.
SO WE HAVE THE ABILITY TO MOVE
FORWARD THROUGH MARCH 8.
AND I BELIEVE IT'S STILL PENDING
THE PRESIDENT'S SIGNATURE BUT
THAT'S MEANT TO BE IMMINENT, FIT
HASN'T ALREADY HAPPENED.
THE HOUSE AND SENATE BILLS FOR
OUR BUDGET REMAIN AS THEY ARE.
THEY WERE PROPOSED TO BE A FLAT
BUDGET FOR NCATS.
AND, AGAIN, DEPENDING HOW
SCENARIOS UNFOLD WILL DETERMINE
WHAT BUDGET ULTIMATELY GETS
APPROVED FOR NCATS.
SO, PLEASE STAY TUNED.
I HOPE TO HAVE A BROADER UPDATE
FOR YOU IN MAY.
OKAY.
SO NOW ON TO SOME SORT OF OTHER
NEWS AND ACTIVITIES.
AT OUR LAST MEETING TOO I
MENTIONED THERE WERE A FEW
TOPICS THAT THE ADVISORY
COMMITTEE TO THE DIRECTOR HAD
BEEN TAKEN UP AND EVALUATING.
IN DECEMBER OF LAST YEAR THEY
BOTH HAVE PUT FORWARD THEIR
RECOMMENDATIONS ON THESE TWO
TOPICS.
SO ONE WAS REENVISIONING THE
NIH-SUPPORTED POSTDOCTORAL
TRAINING PROGRAM.
AND THEIR CHARGE WAS TO
EVALUATE, ASSESS, REVIEW, AND
CONSIDER A VARIETY OF THE WAYS
WE COULD EXPLORE TO, NUMBER ONE,
LOOK AT SHORTAGE OF PhDs,
AND NUMBER TWO, THINK ABOUT
SCOPE AND PERSISTENCE OF THE
ISSUE OF GETTING MORE Ph.D.s
INTO THE FOLD AND TRAINING THAT
MAY GO BEYOND ACADEMIA,
CONSIDERS THE FULL BREADTH OF
TRAINING ACTIVITIES, TO SUPPORT
MORE OF A WORK/LIFE BALANCE,
SOME THINGS THAT WERE DISCUSSED.
THE QR CODE ON THE RIGHT IS THE
REPORT OF RECOMMENDATIONS BUT I
WANTED TO HIGHLIGHT THOSE HERE.
SIX RECOMMENDATIONS WERE PUT
FORWARD.
ONE WAS TO INCREASE PAY AND
BENEFITS FOR NIH-SUPPORTED
POSTDOCTORAL SCHOLARS.
TWO WAS TO CREATE AND EXPAND
MECHANISMS TO SUPPORT FULL
TALENT POOL OF POSTDOCTORAL
SCHOLARS.
THIRD WAS TO FACILITATE
TRANSITION OF POSTDOCTORAL
SCHOLARS INTO THEIR NEXT CAREER
STAGE, HERE AGAIN THINKING ABOUT
ROLES BEYOND ACADEMIA.
AND THEN THE THIRD WHICH I THINK
IS REALLY IMPORTANT IS TO
PROMOTE TRAINING AND
PROFESSIONAL DEVELOPMENT OF
POSTDOCTORAL SCHOLARS AND THEIR
MENTORS.
I REALLY LIKE THE CONCEPT HERE
OF NOT JUST THINKING ABOUT
POSTDOCTORAL SCHOLARS BUT ALSO
MENTORS.
IT'S THAT DYNAMIC THAT'S SUPER
IMPORTANT AS WELL.
NUMBER FIVE, SUPPORT SAFE AND
DIVERSE PERSPECTIVES AND
RESEARCH ENVIRONMENTS WITHIN
INSTITUTIONS.
AND SIX, TO IMPROVE MEANS TO
MEASURE AND SHARE CAREER
PROGRESSION FOR POSTDOCS.
THESE ARE THE SIX
RECOMMENDATIONS THAT WE'RE NOW
CONSIDERING AT THE NIH TO
UNDERSTAND HOW WE CAN THEN
IMPLEMENT THEM AND MOVE FORWARD.
STAY TUNED ON THESE.
I'M SURE IF YOU HAVE POSTDOCS
WITHIN YOUR INSTITUTIONS THAT
YOU'RE ALREADY PAYING ATTENTION
TO THESE BUT HOPE THIS WILL ADD
SOME VALUE TO THAT.
THE NEXT TOPIC THAT WAS
DISCUSSED LAST YEAR FOR THE
ADVISORY COMMITTEE TO DIRECTOR
WAS ON CATALYZING DEVELOPMENT
AND USE OF ALTERNATIVE METHODS
TO ADVANCE BIOMEDICAL RESEARCH,
SO-CALLED NAMs, NEW APPROACH
METHODS, NOVEL APPROACH
METHODOLOGIES, A COUPLE ACRONYMS
ARE USED FOR THIS.
AND CHARGE FOR THIS GROUP WAS TO
IDENTIFY THESE METHODS, TO
ASSESS STRENGTHS AND WEAKNESSES
FOR A VARIETY OF ACTIVITIES THAT
WE DO IN THE BIOMEDICAL
ECOSYSTEM, CHARACTERIZE AND
UNDERSTAND WHAT CONDITIONS THEY
CAN BE USED FOR RECOGNIZING IT'S
NOT ABOUT NECESSARILY REPLACING
BUT ACTUALLY PROVIDING OTHER
MEANS BY WHICH TO STUDY DISEASE
PROCESSES THAT MAY REDUCE THE
ABILITY -- REDUCE OUR USE OF
ANIMAL MODELS AS WELL,
RECOGNIZING WE'RE PROBABLY NOT
GOING TO BE ABLE TO REPLACE THEM
WHOLLY.
THAT'S NOT REALLY THE GOAL.
THE GOAL IS TO UNDERSTAND HOW WE
CAN COMPLEMENT THEM.
SO, THIS GROUP CAME TOGETHER TO
THINK ABOUT THESE DIFFERENT
IDEAS AND QR CODE IS SHOWN HERE
FOR THEIR REPORT.
AND I WILL GIVE YOU JUST A
LITTLE BIT OF OVERVIEW OF THOSE
RECOMMENDATIONS.
I ALSO WANTED TO LET YOU KNOW
THAT FOR THIS PARTICULAR
ACTIVITY WE HAD SPECIFIC PEOPLE
FROM THE FDA, EPA, ALSO FROM
NCATS, BECAUSE NCATS HAS BEEN A
STRONG LEADER IN THIS SPACE AND
WE WILL CONTINUE TO DO SO.
I WANTED TO LET YOU KNOW WHO WAS
ON THIS PARTICULAR PROGRAM AND A
VARIETY OF EXTRAMURAL MEMBERS
FROM THE COMMUNITY WITH A
VARIETY OF DIFFERENT EXPERIENCE
AND EXPERTISE THAT WERE BROUGHT
TO THE TABLE.
SO THIS WAS A FANTASTIC GROUP
THAT REALLY PULLED TOGETHER,
THREE DIFFERENT KIND OF NAMs
THEY ARE CONSIDERING.
THESE ARE CELL-FREE METHODS AND
CHEMICAL GENETICS, FOR EXAMPLE.
IN VITRO METHODS ARE CULTURED
CELLS, iPS AND
MICROPHYSIOLOGICAL SYSTEMS OR
3D CHIPS AND BIO.
AND IN SILICO.
HOW WE CAN USE THESE IN BASIC
RESEARCH, TRANSLATE INTO
PRACTICE.
THESE ARE RECOMMENDATIONS THEY
PUT FORWARD TO PRIORITIZE
DEVELOPMENT OF USE, ESTABLISH
RESOURCES AND INFRASTRUCTURE,
PROMOTE DISSEMINATION, INVEST IN
TRAINING TO BOLSTER THE NEXT
CADRE OF SCIENTISTS, AND TO
DEVELOP INTERDISCIPLINARY TEAMS
AND PROMOTE SOCIAL
RESPONSIBILITY IN THE CREATION
AND DEPLOYMENT OF NAMs ACROSS
THE RESEARCH LIFE CYCLE.
I THINK THIS IS A REALLY
IMPORTANT ONE AS WELL.
AND THEN TO SUPPORT AND MAINTAIN
COORDINATED INFRASTRUCTURE TO
THEN MAKE THESE NAMs BEING
USED IN A RESPONSIBLE WAY, TO
CONTINUE FURTHER DEVELOPMENT.
ONE OF THE UNIQUE ASPECTS OF
THIS WORKING GROUP WE WERE
CONSIDERING AND STILL ARE
CONSIDERING A COMMON FUND
APPROACH TO LOOK AT WHAT WE'RE
CALLING COMPLEMENTARY, WHICH IS
ESSENTIALLY COMPLEMENT ANIMAL
RESEARCH IN EXPERIMENTATION, A
TORTURED ACRONYM.
IF YOU GOOGLE IT, YOU CAN FIND
IT WELL IN THE GOOGLE SPACE.
THAT'S ALWAYS GOOD.
WE WERE WORKING ON THIS WHEN THE
ACD WAS TALKING ABOUT THESE
ISSUES, THAT HELPED US I THINK
SOLIDIFY SOME AREAS WE MIGHT BE
ABLE TO GO INTO.
AS WE'RE THINKING ABOUT PLANNING
FOR THIS ACTIVITY, IF IT DOES
CONTINUE TO MOVE FORWARD, WE
PULLED TOGETHER ROUNDTABLES
ASKING INDUSTRY AND ACADEMIC
PARTNERS, NON-GOVERNMENT AND
ORGANIZATIONAL REPRESENTATIVES
AS WELL AS FDA PARTNERS TO COME
TOGETHER AND TELL US WHAT THEY
WERE SEEING IN THE SPACE AND HOW
WE MIGHT BE ABLE TO HELP MOVE
THIS FORWARD.
IN ADDITION TO PART OF THIS
LANDSCAPE IS DOING SORT OF A
LANDSCAPE ANALYSIS BUT ALSO
IDEATION PRIZES.
SO WE HAD A CHALLENGE OR PRIZE
COMPETITION TO ASK PEOPLE TO
SUBMIT THEIR IDEAS FOR HOW WE
COULD USE COMPLEMENTARY IN A WAY
THAT COULD PUSH THE FIELD
FORWARD.
AND SO I'M EXCITED TO SEE WHERE
THIS GOES BUT I THINK ULTIMATELY
IT'S HOW WE PULL TOGETHER THOSE
THREE DIFFERENT KINDS OF
COMPLEMENTARY APPROACHES IN
THOSE NAMs, AND INTEGRATE THEM
TOGETHER TO MAKE THEM EVEN
GREATER THAN THE -- THE WHOLE
GREATER THAN THE SUM OF THEIR
PARTS, AND I THINK THAT WOULD BE
A REALLY GOOD ADVANCE FOR THE
BIOMEDICAL COMMUNITY.
SO PLEASE STAY TUNED ON THAT.
NOW I MENTIONED TOO THE DEEP
INTERACTIONS WITH THE FDA AS
WE'VE BEEN TALKING ABOUT THAT
IDEA.
BUT IN PARALLEL, NCATS
SPECIFICALLY, I SPECIFICALLY
REALLY WANTED TO MAKE SURE WE'RE
STRENGTHENING OUR PARTNERSHIPS
WITH FDA TOO BECAUSE OF OUR
PLACE IN THE TRANSLATIONAL
SCIENCE PIPELINE, SPECTRUM,
PRE-CLINICAL AND CLINICAL
ACTIVITY IS -- FDA ARE REALLY
OUR PARTNERS.
SO WE'VE WORKED WITH THEM ON A
VARIETY OF TOPICS AND WE WANT TO
CONTINUE TO DO SO.
ONE TOPIC WAS AROUND REALLY
TALKING ABOUT IDEAS IN GENERAL
BUT THE OVERARCHING THEME FOR
THIS WAS GENE THERAPY AND GENE
EDITING, WORKING WITH CBER
COLLEAGUES, INCLUDING RELEVANT
AREAS OF INFORMATICS IN THIS
SPACE, EVEN THE NAMs AS WELL.
SO THIS IS ONE AREA WE'VE BEEN
WORKING ON.
ANOTHER TOPIC OF INTERACTION,
CONTINUING ON THE NAM THEME IS
THINKING ABOUT THE TOXICOLOGICAL
RESEARCH THAT WE ALSO THINK IS
VERY IMPORTANT.
SO WE -- A FEW OF US WENT OUT TO
AR-KANSAS, I SAY THAT BECAUSE
I'M FROM KANSAS, SORRY, BUT I
GUESS I SHOULD SAY ARKANSAS.
AND WE HAD A GREAT MEETING WITH
TUCKER PATTERSON AND HIS TEAM
FROM NCTR, OPPORTUNITIES TO
ADVANCE HUMAN CELL-BASED MODELS
FOR SAFETY AND EFFICACY AND
REALLY WHAT THEY MIGHT NEED TO
HELP US THINK THIS THROUGH.
NCTR IS SUCH A VALUABLE PARTNER
HERE BECAUSE A LOT OF THE WORK
THEY DO IS IN ANIMAL MODELS, SO
IT WILL BE CRITICAL TO BE ABLE
TO UNDERSTAND, YOU KNOW, WHAT
THEY ARE SEEING FROM THEIR END
ON THE ANIMAL MODEL SPACE AND
INCORPORATING THEN THESE NOVEL
APPROACH METHODOLOGIES TO
COMPARE.
SO THIS IS A REALLY IMPORTANT
PARTNERSHIP THAT WE HAVE.
COMING SOON, IN MAY, WE'RE
HOLDING ANOTHER MEETING TO TALK
ABOUT NATURAL HISTORY STUDIES.
AND ALSO HE DISEASE REGISTRIES
IN RARE DISEASE TREATMENTS.
THE IDEA HERE IS TO THINK ABOUT
THE ROLE AND DESIGN OF THESE
REGISTRIES AND HOW THEY COULD BE
USED VERY SPECIFICALLY IN SORT
OF CLINICAL ASSESSMENTS,
ENDPOINTS, BIOMARKERS, ET
CETERA.
THIS WILL BE AN IMPORTANT
MEETING I THINK, ESPECIALLY FOR
OUR RARE DISEASE COMMUNITY.
SO STAY TUNED FOR THAT.
AND REGISTRATION IS NOT YET
OPEN, BUT HOPEFULLY WE CAN FIND
THAT ON THE WEBSITE AND MAKE YOU
AWARE OF THAT.
ALL RIGHT.
I WANT TO ALSO TALK ABOUT SOME
OF THE INNOVATIONS AND IMPACTS
THAT NCATS HAS HAD SINCE WE LAST
SPOKE.
AND SORT OF ON A CONTINUOUS
THEME HERE ABOUT THESE NOVEL
APPROACH METHODOLOGIES, WE HAD A
MEETING ON WHAT WE CALL I3D RARE
WORKSHOP TO DEVELOP 3D
BIOPRINTED TISSUES, TO CONTINUE
A PRECISION MEDICINE PLATFORM OF
STEM CELL-DERIVED TISSUES FROM
PARTICULAR RARE DISEASE
PATIENTS.
AND THINK ABOUT HOW WE CAN USE
THESE TO ACCELERATE DEVELOPMENT
OF THERAPEUTICS FOR RARE
DISEASES IN THIS WAY, SO THE
WORKSHOP BROUGHT TOGETHER A
VARIED OF STAKEHOLDERS WHO COME
FROM DIFFERENT AREAS OF THE
BIOMEDICAL COMMUNITY, AND
DISCUSSED GAPS, BOTTLENECKS,
CHALLENGES WE SEE AT THE FORE OF
HOW WE CAN MOVE FORWARD IN THIS
SPACE ESPECIALLY WHEN THERE
AREN'T MANY ANIMAL MODELS FOR
RARE DISEASES.
SO THIS BECOMES PARTICULARLY
IMPACTFUL IF WE'RE ABLE TO THINK
MORE GENERALLY ABOUT THIS TO
APPLY THEM TO AS MANY OF THESE
TYPES OF MODELS THAT WE CAN DO
PERHAPS IN THE FUTURE.
THIS WAS A RECORDED EVENT, SO
I'M COPIED THE QR CODE FOR THE
YouTube IF YOU'RE INTERESTED
IN VIEWING THAT.
I WANTED TO SHOW YOU TOO JUST
SORT OF POWER THAT WE'RE
THINKING ABOUT IN TERMS OF 3D
MODELS THAT HAVE BEEN EVALUATED
AT NCATS.
THEY HAVE DEVELOPED TISSUE
MODELS FOR GENE THERAPY THROUGH
OUR PAVE-GT PROGRAM, WE'RE
USING -- WE'RE LOOKING AT TWO --
WELL, I SHOULD SAY FOUR REAL
DISEASES FOR GENE THERAPY HERE
FOR THE PAVE-GT PROGRAM AS A
PILOT FOR THIS, TWO DISEASES ARE
IN ORGANIC ACIDEMIA, AND TWO ARE
NEUROMUSCULAR JUNCTION, AAV
VECTORS WILL BE MADE FOR THOSE
GENES THAT WILL SERVE AS SORT OF
GENE REPLACEMENT.
SO WE WANTED TO CREATE LIVER
SPHEROIDS FOR ACIDEMIA GENE
THERAPY AND WE RECEIVED CELLS
FROM A PATIENT FROM PITTSBURGH
AND CREATED ORGANOIDS FOR THIS
PARTICULAR ACTIVITY, LOOKING AT
HOW TO INFUSE VECTORS TO LOOK
AT FUNCTIONAL READOUTS STARTING
WITH ONE TISSUE BUT ULTIMATELY,
YOU KNOW, THE IMPACT OF WHAT IT
MIGHT HAVE ON OTHER SYSTEMS CAN
BE EVALUATED IN THE FUTURE.
WE LOOKED AT NEUROMUSCULAR
JUNCTIONS.
IF YOU LOOK CLOSELY YOU CAN SEE
THAT MUSCLE FIBER TWITCHING, AND
HERE AGAIN WE USE iPS CELLS TO
CREATE CELL TYPE THAT WOULD LOOK
AT CONGENITAL MYASTHENIC
SYNDROMES, TO ASSESS HOW THEY
COULD BE USEFUL FOR LOOKING AT
SAFETY AND TOXICITY AND EFFICACY
FOR THESE DISEASES IN THE
FUTURE.
IN ANOTHER SORT OF ACTIVITY
WE'RE ALSO LOOKING AT THE SPACE
OF LARGE LANGUAGE MODELS, AND WE
HAD SORT OF A HYBRID MEETING IN
NOVEMBER THAT BROUGHT ERIC TOPOL
AS A KEYNOTE SPEAKER TO TALK
ABOUT UNITS OF USING LLMs.
THIS IS A BIG TOOL.
EVEN IN ITS EARLY PHASES IT'S
QUITE POWERFUL.
WE WANTED TO MAKE SURE WE'RE
APPROACHING IT IN A WAY THAT
MAKES SENSE FOR US IN THE
RESEARCH WE WANT TO DO.
SO THE FOLKS ON THE LEFT, MIKE,
JOSH, SAM AND DAVID, PULLED A
GROUP TOGETHER.
THE QR CODE IS THE VIDEO CAST.
WHAT CAME OUT WAS EVOLVING NEEDS
WE WANT TO PAY ATTENTION TO AS
WE GO FORWARD, COMPUTATIONAL
RESOURCES, DATA SOURCES,
TRUSTWORTHINESS OF THE DATA, CAN
WE TRUST THESE LOMs, HOW DO WE
NAVIGATE THAT AND AND EVALUATE
THAT.
WHAT ARE BEST PRACTICES,
ESPECIALLY WHEN IT COMES TO
REGULATORY POLICY
CONSIDERATIONS.
IMPORTANT IS ETHICS, DIVERSITY
AND INCLUSION, MAKING FOLKS
AWARE OF THE VALUE OF THESE
SORTS OF TYPES OF DATA THAT CAN
BE USED.
SO, WE'RE EXCITED ABOUT THIS,
YOU KNOW, CONTINUING TO HAVE
THESE CONVERSATIONS, AND THIS IS
ALSO TRANSLATED INTO SOME OF THE
WORK THAT LITCOIN HAS BEEN
DOING, THIS EXCITING NCATS
PROGRAM OUT OF CHRISTINE COLVIS'
OFFICE, TYLER BECK IS ONE OF THE
LEADERS.
ONE TO INCENTIVIZE DATA AND
KNOWLEDGE SHARING GIVING AUTHORS
A WAY TO PUBLISH VALUABLE
RESEARCH THAT FALLS OUTSIDE THE
CURRENT PUBLISHING PARADIGM.
SO THAT MEANS NEGATIVE RESULTS,
FOR EXAMPLE, REPLICATION
STUDIES, OR OTHER MISCELLANEOUS
FINDINGS YOU MAY NOT THINK ABOUT
A PUBLICATION BUT THIS OFFERS A
WAY TO PERHAPS PUBLISH THOSE
DATA AND MAKE THEM AVAILABLE.
SO, IT ALLOWS US TO BUILD
MACHINE READABLE OR A.I.-READY
KNOWLEDGE BASE TO CONSIDER OUT
OF THE TEXT THAT COULD COME
FROM.
AND I WANTED TO GIVE AN UPDATE
ON SORT OF WHAT I MEAN BY THAT
SECOND BULLET HERE.
SO, IN BUILDING THIS
MACHINE-READABLE A.I.-READY
KNOWLEDGE BASE THESE LARGE
LANGUAGE MODELS CAN HAVE ABILITY
TO IDENTIFY AND SORT OF GRAB
THESE KEY FINDINGS IN THE
SUBMITTED MATERIALS.
SO IT CAN BE PRETTY QUICK AND
ACCURATE, AND FAIRLY CONSISTENT
TOO IN TERMS OF THE
IDENTIFICATION OF SCIENTIFIC
RESEARCH, FINDINGS WITHIN THE
LITERATURE, BUT REALLY AT SCALE.
AND THESE FINDINGS ARE BROKEN
DOWN INTO SUBJECT, OBJECT, AND
RELATION.
AND ASSEMBLED IN THAT WAY INTO
THE MACHINE-READABLE SORT OF
FIELD LEVEL.
BEFORE YOU CAN INCORPORATE THAT
INTO ANY LITCOIN CORPUS WE'RE
THINKING ABOUT, THESE KNOWLEDGE
GRAPHS ARE VALIDATED TO ENSURE
THEIR ACCURATE, TRUSTING THESE
SORTS OF MODELS.
IT ALSO IS BUILT ON TOP -- THIS
ISN'T NEW, BUILT ON TOP OF META.
META CREATED THIS PLATFORM
THAT'S DESIGNED TO FACILITATE
COLLABORATION, INFORMATION
SHARING, IN THE BIOMEDICAL
RESEARCH COMMUNITY.
SO LAVA 2 IS PARTICULARLY
HELPFUL FOR TRANSLATIONAL
SCIENCE AND RARE DISEASES AND
RELATED FIELDS, SO LITCOIN HAS
TAKEN A PAGE FROM THE LLAMA 2
PROGRAM, CREATED A PROGRAM
CALLED HEAL-PACA, FOR THE
HELPING END ADICTION LONG TERM
STUDY, TRANSLATING TO CREATING
THIS LLM HEALPACA FOR ALGORITHMS
AROUND THE HEAL-RELATED WORK.
TO DEVELOP THIS PART OF THE
LITCOIN ALGORITHM FOR HEALPAC,
SUGGESTING HEALPACA MAY
OUTPERFORM CHATGPT IN HUMAN
COMPARISON MODELS AND OUTPUTS.
WE HAD A LOT OF FUN PUTTING THIS
LITCOIN TOGETHER, ALPACA-PUNCH
WITH THIS VISUAL, VISUALS ARE SO
GOOD.
IN ANOTHER AREA TALKING ABOUT
TRANSLATIONAL SCIENCE AND
EDUCATION, AND TRAINING
PROGRAMS, WE HAVE CREATED THESE
EIGHT KEY PRINCIPLES OF
TRANSLATIONAL SCIENCE.
I HAVE EVEN A VISUAL HERE OF
SOME STICKERS AND MAGNETS WE
HAVE.
I'LL MAKE SURE TO GIVE THOSE TO
YOU NEXT TIME WE'RE IN PERSON.
BUT THESE HAVE BECOME REALLY
FANTASTIC TOOLS FOR PEOPLE WHO
ARE THINKING ABOUT TRAINING IN
EDUCATION FOR TRANSLATION
ALSCIENCE, RESONATING WITH EIGHT
KEY PRINCIPLES OF WHAT IT MIPS
MEANS TO BE A TRANSLATIONAL
SCIENTIST.
I'VE BEEN HIGHLIGHTING THESE,
KEY THEMES THROUGHOUT CTSA
MEETING LAST FALL, ADOPTED THIS
AS A WAY OF COMMUNICATING WHAT
THE WORK THAT WE'RE TRYING TO
DO, HOW IT FITS IN THE BROADER
LANDSCAPE OF THESE KEY
PRINCIPLES.
WE'VE ALSO PUBLISHED A
LITERATURE REVIEW ON
TRANSLATIONAL SCIENCE EDUCATION
AND TRAINING ALIGNED WITH THESE
PRINCIPLES.
I PUT THAT QR CODE HERE.
NEXT YEAR WE'LL CONTINUE TO DO
THIS WORK, DEVELOP MORE CASE
STUDIES OF NCATS' SUCCESSES AND
DEVELOP RESOURCES FOR OUR
COMMUNITY.
SO WE'RE PRETTY EXCITED ABOUT
THIS PART TOO.
ALL RIGHT.
I WANT TO MOVE INTO THE AREA OF
WHAT WE'VE CONTINUED TO DO IN
THIS SPACE OF COVID-19.
YOU KNOW, ALTHOUGH WE'RE KIND OF
MOVING ON OUT OF THE COVID-19
REALM, BUT NCATS IS STILL VERY
MUCH LEARNING A LOT FROM THE
WORK THAT WE'VE BEEN DOING IN
COVID.
AND SEE A LOT OF VALUE IN
CONTINUING THAT.
TWO COUNCILS AGO I SHOWED YOU A
PAPER THAT WAS PENNED BY SEVERAL
NCATS AUTHORS HERE, SHOWN HERE
ON PAXLOVID EFFECTIVENESS, USING
N3C DATA, A PAPER REQUESTED FROM
THE COVID-19 RESPONSE TEAM AT
THE WHITE HOUSE, AND HE SAID,
HEY, CAN YOU TAKE A LOOK AT
WHETHER OR NOT -- HOW MANY
PEOPLE HAVE GOTTEN PAXLOVID WHO
WERE ELIGIBLE, HOW MANY HAVEN'T
GOTTEN BUT WERE ELIGIBLE.
WHAT WOULD THE IMPACT HAVE BEEN
HAD THEY RECEIVED PAXLOVID, THAT
WAS THE QUESTION.
THE QR CODE IS ON THE UPPER
RIGHT.
BASICALLY THE FIGURE SHOWN HERE
SHOWS THE UPTAKE OF COVID FROM
VERY RED ZERO TO BLUE VERY HIGH,
AND YOU CAN SEE IN 2021 PAXLOVID
FIRST CAME OUT, IT WASN'T USED
VERY MUCH.
2022 STARTING TO BE EVEN MORE.
2023 AND EVEN NOW, IT'S PROBABLY
NOT USED AT MAXIMAL POTENTIAL.
THIS PAPER IS STARTING TO GET
ATTENTION IN THE PRESS, COVERED
NOW, IT'S A PRE-PRINT STILL BUT
COVERED IN THE "NEW YORK TIMES,"
WASHINGTON POST, USA TODAY, SO A
SHOUT OUT TO JOSH FESSEL WHO
COORDINATED THIS EFFORT, HE SAID
IF YOU CAN TAKE PAXLOVID AND YOU
DO, WITHIN THE RECOMMENDED TIME
FRAME, LIKELIHOOD OF DEATH OR
HOSPITALIZATION ARE
SIGNIFICANTLY REDUCED, THAT'S A
BIG DEAL.
THIS PAPER SHOWED APPROXIMATELY
48,000 DEATHS AND 135,000
HOSPITALIZATIONS AT THE TIME OF
THIS PUBLICATION COULD
POTENTIALLY HAVE BEEN PREVENTED
HAD THE ELIGIBLE PEOPLE TAKEN
THE PAXLOVID.
WE'RE STILL WANTING TO LEARN
ABOUT THE ENTHUSE OF THESE
THERAPEUTIC TOOLS FOR FIGHTING
AND COMBATING COVID-19 BECAUSE
IT'S STILL OUT THERE, THIS IS
IMPORTANT PUBLIC HEALTH KIND OF
WORK.
WE'RE NOW TRANSITIONING TO
LOOKING AT AND CONTINUING EVEN
SOME OF THE ACTIVE SIX STUDIES
THAT WE'RE DOING.
ACTIVE SIX IS ONE OF THE
PROGRAMS THROUGH THE COVID-19
WORK THAT WE'VE BEEN DOING.
AND IT IS A COMPLETELY VIRTUAL
CLINICAL TRIAL, DECENTRALIZED
CLINICAL TRIAL, AND METFORMIN IS
NOW OUR LAST THERAPEUTIC THAT
WE'RE EVALUATING.
WE SAW FROM A COVID OUT CLINICAL
TRIAL CONDUCTED PRIOR TO THIS,
PRIOR TO OUR TAKING IT ON, THAT
THEY SAW BENEFIT OF USE OF
METFORMIN FOR COVID-19.
AND SO IT WAS A SMALL STUDY, WE
WANTED TO EVALUATE THAT IN THE
ACTIVE SIX PLATFORM.
SO SARAH DUNSMORE IS LEADING
THIS SPACE, LOOKING AT PRIMARY
ENDPOINT, TIME TO RECOVERY FOR
THESE ACUTE COVID SYMPTOMS BUT
WE'RE ALSO ABLE TO EVALUATE THE
LONG COVID SYMPTOMS AS WELL
BECAUSE WE WILL HAVE A LONGER
TERM FOLLOW-UP, EXPANDING THIS
PARTICULAR METFORMIN ARM OF THE
ACTIV 6-PLATFORM.
WE'LL BE ABLE TO DO THIS
RANDOMIZED CLINICAL TRIAL.
WE HOPE TO GET TO 3,000
PARTICIPANTS AND BE ABLE TO
EVALUATE THESE PRIMARY ENDPOINTS
AS WELL AS LONG COVID ENDPOINTS.
WE HAVE THE FASTER PROGRAM, N3C
PUBLIC HEALTH ANSWERS TO SPEED
TRACTABLE RESULTS, AN ACRONYM,
HELPS US GET TO ANSWERS FASTER,
WE'RE LOOKING AT DATABASE FROM
ELECTRONIC HEALTH RECORD FROM
PEOPLE WHO STARTED TAKING
METFORMIN AFTER A COVID-POSITIVE
TEST, THAT COMBINED WITH THE
RANDOMIZED CLINICAL TRIAL AS
WELL, TRIAL EMULATION, REAL
TRIAL, WE'LL BE ABLE TO ASSESS
THE VALUE OF THE TRIAL EMULATION
METHODOLOGY USING ELECTRONIC
HEALTH RECORDS TO HELP INFORM
MAYBE HOW WE WANT TO DESIGN AND
SET UP FUTURE CLINICAL TRIALS.
WE'RE EXCITED ABOUT DOING THIS
AND THIS UNIQUE WAY THROUGH
ACTIV-6 AND N3C PLATFORM.
YOU CAN SEE MORE ON THE QR CODE
SHOWN HERE.
METFORMIN, WE HAVE A DATABASE ON
METFORMIN, IF YOU'RE INTERESTED
IN LOOKING AT THAT, HERE IS
WHERE WE HAVE NUMBERS OF PEOPLE
WITHIN N3C, RECORDS THAT ARE
AVAILABLE FOR THIS TYPE OF
ANALYSIS.
AGAIN, THIS WILL BE VERY HELPFUL
FOR US MOVING FORWARD.
I ALSO JUST WANTED TO SAY A NOTE
ON N3C IN AND OF ITSELF.
THIS IS SORT OF LIKE OUR
SNOWSTORM TODAY.
IT'S OVERPERFORMING.
IT HAS REALLY BEEN SUCH A HUGE
SUCCESS, AND REALLY THANK ALL OF
THE NCATS TEAMS WHO HAVE BEEN
WORKING ON THIS.
THERE ARE TOO MANY TO NAME.
SO MANY PEOPLE ON THE EXTRAMURAL
COMMUNITY AS WELL.
BUT CTSA PROGRAM, YOU KNOW, HAD
THIS VISION AS WELL.
BUT IT WAS ALSO RDCRN GROUPS WHO
LAID THE FRAMEWORK FOR PULL
DISPARATE DATA TYPES TOGETHER IN
A WAY, VIRTUAL RESEARCH
RESEARCH INFRASTRUCTURE.
THE RDCRN HAD THE SEEDS TO HAVE
THE TOOLS IN PLACE TO BUILD AND
BE READY FOR THE PANDEMIC, AND
THEN THE CTSA PROGRAM WAS REALLY
READY TO JUMP IN AND EVEN ADD
MORE VALUE TO THAT.
SO THIS HAS REALLY BEEN ACROSS
THE BOARD NCATS APPROACH, I
WANTED TO SAY HOW PROUD I AM OF
EVERYONE WHO HAS WORKED ON THIS
TO CREATE SUCH A NATIONAL
VIRTUAL RESEARCH INFRASTRUCTURE
WITH GOVERNANCE, WITH
INTEROPERABILITY,
DE-IDENTIFICATION, BEST
PRACTICES, IMPORTANT FOR
EVALUATING DATA.
I WANTED TO GIVE YOU A SENSE OF
WHERE WE'RE GOING.
WE STARTED WITH COVID-19 FOR
N3C, BUT NOW WE'RE THINKING
ABOUT IN ADDITION TO IF THERE
ARE WAYS TO CONTINUE THAT WE
WANT TO EXPLORE THAT.
BUT ALSO WE'RE THINKING ABOUT
WAYS TO EXPAND IT INTO NON-COVID
SPACES, AND WORKING WITH THE
COMMUNITY TO SEE BEST WAYS OF
DOING THAT.
WE'VE ESTABLISHED THIS IDEA OF
BEING ABLE TO COMPARE ONE EHR
FROM ONE HOSPITAL TO ANOTHER EHR
IN ANOTHER HOSPITAL, THAT ARE
COMPLETELY DISPARATE.
AND NOW WE CAN CONVERT THOSE
MODELS ONTO ONE ANOTHER THROUGH
WHAT WE CALL CODE MAPPING
SERVICES.
THIS IS REALLY KEN GERSING IS
MASTERFUL AT THIS IDEA, HAS
REALLY LED THIS WORK.
BUT WE CAN BASICALLY TAKE ANY OF
THESE MODELS AND CONVERT THEM TO
ANOTHER MODEL, WITHOUT ANY
TROUBLE, MAP THEM ONTO FHIR
RESOURCES TOO.
THIS IS COLLABORATION, DATA
LINKAGE, AND HELPING US
STREAMLINE FDA SUBMISSIONS AND
PHARMACOVIGILANCE, ENSURING WE
HAVE A CONTROLLED VOCABULARY
TRANSFORM FOR VERY DISPARATE EHR
DATA SOURCES.
EHR IS MESSY, THERE NEEDS A LOT
OF WORK IN THIS SPACE BUT I
THINK IF WE CAN ELEVATE AND
MATURE THE MESSINESS, SO TO
SPEAK, OF EHRs WE'RE VERY MUCH
READY TO CONTINUE TO WORK
THROUGH THOSE IN THESE CODE
MAPPING SERVICE WAYS SO WE CAN
COMPARE AND INTEROPERATE THE
SCIENTIFIC QUESTIONS THAT WE
HAVE.
SO, N3C IS A CENTRALIZED
RESOURCE, CENTRALIZED ANALYTICS.
WE'RE NOW THINKING ABOUT MOVING
INTO A TENANT MODEL TESTING FOR
FEASIBILITY.
ESSENTIALLY WHAT THIS MEANS IT
INCORPORATES A FEDERATED
APPROACH, DATA LIVE AT ACADEMIC
MEDICAL CENTER, AND THEN IF THEY
ARE INTERESTED IN RESEARCH
QUESTION THEY CAN THEN DELIVER
THEIR DATA TO AN ENCLAVE LIKE
N3C AND HAVE A RESEARCH
QUESTIONS OTHERS MIGHT WANT TO
ADD THEIR DATA TO AS WELL SO WE
CAN CREATE THEN THESE TENANT
MODELS OF A PARTICULAR RESEARCH
QUESTION THAT PEOPLE CAN USE AND
EVALUATE.
SO THAT'S THE KIND OF MODEL
WE'RE THINKING ABOUT AND
PILOTING AS WE SPEAK TO
DETERMINE FEASIBILITY, SECURITY,
ET CETERA.
SO STAY TUNED ON THIS.
WE'RE TRYING TO TEST THE WATERS
ON IT.
ALL RIGHT.
I'M GOING TO MOVE INTO THE RARE
DISEASE SPACE NOW.
AND, YOU KNOW, JANUARY IS A
PRECURSOR TO THE FEBRUARY RARE
DISEASE DAY SO THIS IS ALWAYS A
FUN TIME TO BE PREPARING FOR ONE
OF MY FAVORITE MEETINGS.
WE HAVE ARTIFICIAL INTELLIGENCE
THIS YEAR, ITS ROLE IN RARE
DISEASES, SO BUILDING OFF
DISCUSSIONS WE HAD EARLIER.
RECENT APPROVALS IN GENE
THERAPY, ENGAGING THE RARE
DISEASE COMMUNITY TO HEAR FROM
THEIR STORIES.
I CAN'T THANK ALICE, MEERA AND
AINSLIE ENOUGH, AND OF COURSE
NOW THE DIRECTOR OF DRDRI.
REGISTER IF YOU HAVEN'T.
THE QR CODE IS THERE.
I HOPE TO SEE YOU.
IN OTHER RARE DISEASE NEWS, WE
HAVE TWO BASKET TRIALS OF SORT
OF OUR SHARED MOLECULAR
ETIOLOGY GROUP, TREATING MORE
THAN ONE DISEASE AT A TIME
GROUP.
TWO STUDIES HAVE A SAFE TO
PROCEED LETTER FROM THE FDA,
WE'RE EXCITED ABOUT THAT.
AND THEN A QR CODE TO AN SBIR
PROGRAM OF THE SAME VEIN, WANTED
TO MAKE YOU ARE AWARE OF THE
SBIR DUE MARCH 15.
WE'RE TRANSITIONING SOMATIC GENE
CELL PROGRAM IN THE COMMON FUND,
P.J. BROOKS IS OUR LEAD FOR THIS
PROGRAM, ACROSS THE NIH.
AND WE MOVED FROM PHASE 1 TO
PHASE 2, NOW HOPING TO START TO
DRIVE SOME GENE EDITING
ACTIVITIES MORE TOWARDS CLINICAL
DEVELOPMENT, WE'RE VERY EXCITED
ABOUT KICK OFF THIS PARTICULAR
PART OF THE PROGRAM.
ONE OF THE CHALLENGES FOR THE
FIRST PHASE TRANSITION INTO THE
SECOND IS HERE, PROGRAMMABLE
DELIVERY SYSTEMS, WHAT ARE
EFFICIENT WAYS TO DELIVER GENE
THERAPIES, THEN ALSO LOOKING AT
NON-VIRAL DELIVERY ACROSS THE
BLOOD-BRAIN BARRIER, VERY HARD
TO GET TO TISSUES.
WE HAD 30 WINNERS FOR PHASE 1
CHALLENGE, NOW PHASE 2 IS
UNDERWAY.
THE INFORMATION WEBINAR IS THE
25th OF JANUARY, EVERYTHING
ELSE WILL FOLLOW SUIT.
QUICKLY, SOME UPDATES FROM THE
DPI PERSPECTIVE.
WE HAVE A VARIETY OF REGULATORY
MILESTONES FROM OUR
TRANSLATIONAL DEVELOPMENT
BRANCH.
A DRUG APPROVAL TO TREAT
DUCHENNE MUSCULAR DYSTROPHY.
WE'RE NOT THE SOLE GROUPS
WORKING IN THIS SPACE BUT HAVE
CONTRIBUTED IN SIGNIFICANT WAYS
TO THESE ACTIVITIES.
WE HAVE SAFE-TO-PROCEED
TREATMENTS FROM THE FDA, WE'VE
HELD PRE-IND MEETINGS WITH FDA,
HAVE OTHER APPROVED DESIGNATIONS
FROM THE FDA FOR A VARIETY OF
THESE RARE DISEASE TREATMENTS.
SO JUST A LITTLE SORT OF
AT-A-GLANCE SHOT AT THAT.
ALSO FOR OUR INTRAMURAL
TRAINING, I MENTIONED SOME WORK
GOING ON IN OUR TRAINING
PROGRAMS, BUT CERTAINLY WE'VE I
THINK VERY MUCH MATURED IN OUR
INTRAMURAL TRAINING SPACE TOO.
BELEN HURLE IS FANTASTIC, AND
MARCUS HODGES IS LEAD, BOTH
INCREDIBLE ASSETS AT NCATS AND
OUR TRAINEES.
THEY WORK ON COURSES, WORKSHOPS,
GRANT WRITING, GETTING THEM
EXPOSED TO A VARIETY OF CAREERS,
ET CETERA.
HOLD RETREATS, THINGS OF THAT
NATURE.
AND ALSO MAKING SURE THAT THEY
ARE EXPOSED TO MEETINGS, TO
PROFESSIONAL SOCIETIES TO FOSTER
A DIVERSE SCIENTIFIC WORK FORCE,
SO EXCITED ABOUT THE DIRECTIONS
OUR INTRAMURAL TRAINING PROGRAMS
ARE GOING IN AS WELL.
I WANT TO END, MY LAST TWO
SLIDES, ON STRATEGIC PLANNING
PROCESS.
WE TALKED ABOUT THIS AT OUR LAST
MEETING.
WE'RE NOW IN SORT OF THIS
IN-BETWEEN PHASE.
WE'VE DONE A LOT OF THE
ROUNDTABLES, GATHERED FEEDBACK,
LAST TIME WE SPOKE WE WERE AT
THE STAGE OF REQUEST FOR
INFORMATION THAT WE WERE WANTING
YOUR FEEDBACK ON.
SO WE'VE GATHERED ALL OF THAT
INPUT FROM ALL THE ROUNDTABLES,
FROM ALL THE RESPONSES TO THE
RFI.
AND SO WE'RE NOW AT THIS POINT
OF DRAFTING THE STRATEGIC PLAN,
AND WE PLAN TO DISCUSS THIS MORE
DEEPLY IN OUR MAY COUNCIL BUT I
DIDN'T WANT YOU TO THINK I
FORGOT ABOUT THIS STRATEGIC
PLAN.
AND OBVIOUSLY, IF THERE'S
ANYTHING THAT YOU ARE CURIOUS
ABOUT OR MAYBE FORGOT TO SAY WE
SHOULD INCLUDE WE'D LOVE TO HEAR
FROM YOU CERTAINLY AS WELL.
SO WE LOOK TO GET THIS, YOU
KNOW, DISCUSSED REALLY MORE OF A
DRAFT STAGE IN MAY, AND WE'LL
FINALIZE THAT, ROLLOUT FOR THE
STRATEGIC PLAN .
WE'RE ALMOST TOUCHING THIRD
BASE, WE'RE ON THE HOME STRETCH,
ROUNDING TOWARDS HOME.
OKAY.
THIS IS JUST A VISUAL OF WHERE
WE IN THE DRAFT STRATEGIC GOALS
WE'RE WORKING TOWARDS.
YOU'VE SEEN THIS SLIDE BEFORE SO
I WON'T DWELL ON IT BUT WANTED
TO REMIND YOU ABOUT THAT.
WITH THAT, YOU KNOW, JUST A
SUMMARY HERE IS WE'RE STILL
WAITING, HOW WE'RE GOING TO HAVE
A 2024 BUDGET, BUT WE'RE
OPERATING UNDER A C.R.
WE'VE HAD A VARIETY OF IMPACTFUL
ADVANCES, AND ESPECIALLY IN THE
DATA SCIENCE SPACE, AND GENE
THERAPY, GENE EDITING SPACE.
AND REALLY SOLIDIFYING AND
DEEPENING OUR INTERACTIONS WITH
THE FDA AS WELL.
OUR STRATEGIC PLAN IS ON TRACK
FOR EARLY 2024 DRAFT PLAN, MORE
ABOUT THAT LATER.
AND OF COURSE UPDATES TODAY, DR.
BERTAGNOLLI WILL SPEAK ABOUT HER
VISION AND, YOU KNOW, AS A NEW
DIRECTOR I'M REALLY EXCITED
YOU'RE ABLE TO HEAR FROM HER,
ONE OF THE FIRST TIMES.
I'M EXCITED TO HEAR FROM HER AS
WELL.
DR. BALAKRISHNAN WILL TALK ABOUT
THE OFFICE OF STRATEGIC
ALLIANCE, AND DR. TAGLE SPECIAL
INITIATIVES.
I OPEN IT UP FOR SUGGESTIONS.
>> WOW.
>> THANK YOU.
SERGIO?
>> WHAT A WONDERFUL PRESENTATION
AND REVIEW.
ONE COMMENT THAT I SO MUCH
APPRECIATE THAT YOU INCLUDE THE
QR CODES IN EACH OF THE SLIDES.
IT'S VERY WELL DONE.
AND I HAVE BEEN CURIOUS ENOUGH
TO CLICK.
LIKE IN THE PAPER THAT YOU
PUBLISHED ON EDUCATION, YOU
KNOW, WITH JCPS, SO IT IS A
WONDERFUL IDEA.
AND VERY HELPFUL AS WELL.
SO THANK YOU.
>> THANK YOU.
YOU KNOW, WHEN WE WENT INTO THE
PANDEMIC, I THINK WE GOT USED TO
PUTTING IN LINKS THEMSELVES.
I REALIZED IT DIDN'T MAKE SENSE.
PEOPLE COULDN'T CLICK ON THEM.
SO, THIS WAS A GOOD WAY TO GET
THAT INTERACTION AND MAKE SURE
THAT EVERYONE HAD THE MATERIALS
AT THE READY.
SO THANK YOU.
HAPPY FOR THE FEEDBACK.
>> YEAH, NO, VERY -- ACTUALLY I
WAS GOING TO ASK IF YOU CAN
SHARE THE SLIDES SO I CAN CLICK
ON SOME THAT I MISSED.
>> YES, ABSOLUTELY.
ABSOLUTELY.
HAPPY TO.
>> SECOND THING IS THAT -- AND
I'M GLAD THAT YOU TOUCHED BASED
A LITTLE BIT ON THE STRATEGIC
PLANNING AS TO WHERE YOU HAVE
BEEN AND WHERE YOU ARE CURRENTLY
AND WHAT ARE THE NEXT STEPS.
ARE YOU PLANNING TO ASK FOR
PUBLIC COMMENTS WHEN THE DRAFT,
STRATEGIC PLANNING IS AVAILABLE?
I WOULD ENCOURAGE THAT, AND I
WOULD ENCOURAGE, YOU KNOW, TO
REALLY GET IN PARTICULAR
COMMUNITIES, YOU KNOW, CBOs,
COMMUNITY MEMBERS, ADVISORY
BOARDS, ESPECIALLY ADVISORY
BOARDS, ACROSS CTSAs TO WEIGH
IN.
YOU'VE ALREADY DONE A FANTASTIC
JOB WITH LISTENING TOURS.
AND WHICH IS TERRIFIC.
I ENCOURAGE YOU, IF THAT CAN BE
FITTING.
>> THANK YOU FOR THAT, SERGIO.
MEREDITH IS THE ONE LEADING OUR
STRATEGIC PLANNING ACTIVITIES, I
COULD ANSWER THIS BUT I'M GOING
TO ALLOW MEREDITH TO DO THIS.
>> HELLO.
THANK YOU, JONI.
AND THANK YOU, DR. AGUILAR.
SO, YES, A COUPLE THINGS TO FILL
IN.
ONE, WE DID DO RFI ON THE DRAFT
FRAMEWORK.
SO, WE DID HAVE A REQUEST FOR
INFORMATION AND GOT OVER 50
COMMENTS BACK FROM FOLKS ABOUT
THE DRAFT GOALS AND, YOU KNOW,
KIND OF WHERE THE FRAMEWORK WAS
AT THAT POINT.
THAT WAS -- WE POSTED THAT I
BELIEVE BACK IN SEPTEMBER, IT
RAN THROUGH NOVEMBER.
AND THE OTHER PLAN WE HAVE, AS
YOU MAY HAVE SEEN IN JONI'S
TIMELINE, SHE'S GOING TO SHARE
THE SLIDES, WE'RE DRAFTING THE
PLAN AND THEN IN GENERAL WHAT
MOST NIH I.C.s DO IS POST A
DRAFT OF THE PLAN PUBLICLY ON
THEIR WEBSITE.
AND THEN THAT GIVES, YOU KNOW,
ALL CONSTITUENTS THE OPPORTUNITY
TO TAKE A LOOK AT IT AND GIVE US
ANY FEEDBACK RIGHT BEFORE WE
FINALIZE IT.
SO, AS JONI WAS INDICATING, OUR
PLAN IS TO -- WE'RE STILL IN THE
BUSY WRITING STAGES RIGHT NOW SO
WE'RE NOT EXACTLY SURE HOW ALL
OF THE DUCKS ARE GOING TO
TOTALLY LINE UP BUT OUR PLAN IS
TO HAVE SOMETHING VERY
SUBSTANTIVE TO DISCUSS WITH YOU
ALL IN MAY, AND THEN WE WILL
LIKELY POST IT RIGHT AFTER THAT,
DEPENDING ON THE FEEDBACK THAT
WE GET FROM YOU.
THAT'S OUR TENTATIVE GAME PLAN.
>> I THINK MAYBE, MEREDITH, WHAT
WE WOULD THINK ABOUT THEN IS
ONCE WE DO POST THAT WE COULD
CONSIDER SORT OF THE RFI BUT I
THINK WE MIGHT HAVE BETTER
OUTREACH IF WE DO A DIRECTOR'S
MESSAGE THAT SAYS --
>> CORRECT.
>> AND THEN DO OUR SIMILAR
OUTREACH TO ALL OF THE PEOPLE
WHO JOINED OUR ROUNDTABLES, LIKE
THE ADVISORY BOARDS AT THE
CTSAs, AND MAKE SURE THAT
EVERYBODY HAS ACCESS TO THAT.
SO, I THINK WE'LL DO SOMEWHAT,
AGAIN, OF A WARM TOUCH, A
PERSONAL TOUCH TO THOSE GROUPS.
>> YEP.
>> TO LET THEM KNOW WHAT WE HAVE
ON OUR WEBSITE IS READY TO BE
REVIEWED AND WE WOULD LOVE TO
HEAR COMMENTS BEFORE FINALIZING
THAT DRAFT.
LONG ANSWER TO YOUR QUESTION,
BUT THE SHORT ANSWER IS YES.
>> ABSOLUTELY.
I WAS GOING TO SAY WE'LL BE VERY
MUCH WORKING WITH THE
COMMUNICATIONS TEAM ON OUR WAYS
TO -- EMILY HAS GREAT WAYS OF
REACHING OUT TO OUR DIFFERENT
CONSTITUENTS, SO WE WILL
DEFINITELY BE COMING UP WITH A
PLAN FOR THAT, FOR DISSEMINATING
AND MAKING SURE PEOPLE ARE AWARE
THAT IT'S OUT THERE.
>> YEAH, NO, THAT'S MUSIC TO MY
EARS.
THANK YOU SO MUCH FOR THAT.
THANK YOU, MEREDITH, FOR THE
DETAILS.
AND JONI, ONE LAST THING.
THERE IS A LOT OF ACTIVITY AND
ATTENTION BEING BROUGHT
NATIONWIDE, AT LEAST WITHIN THE
COMMUNITY ENGAGEMENT FIELD,
ABOUT THE IMPORTANCE OF
TRUSTWORTHINESS.
HOW CRITICALLY IMPORTANT IT IS
FOR OUR INSTITUTIONS AND
AGENCIES TO BE TRUSTWORTHY.
JUST TO GIVE YOU THE HEADS UP
THAT PRINCIPLES OF ENGAGEMENT,
THIRD EDITION, IS READY.
>> YES.
>> AND I'M SURE YOU KNOW ABOUT
IT.
>> YES.
>> THERE IS A PRINCIPAL THING
THAT WAS ADDED THIS NEW EDITION,
WHICH IS ON TRUSTWORTHINESS, AS
A FUNDAMENTAL ELEMENT FOR
ADVANCING HEALTH EQUITY.
AND JUST MY BRIEF COMMENT TO YOU
IS THAT -- AND YOU'RE THE FIRST
PERSON I HAVE HEARD THIS FROM.
YOU TALK ABOUT TRUSTWORTHINESS
OF DATA AS WELL.
THAT WAS FASCINATING FOR ME TO
HEAR.
BUT I WOULD BE VERY INTERESTED
IF IN THE LISTENING SESSIONS
THAT YOU HAVE, ESPECIALLY WITH
COMMUNITIES AND ADVISORY -- WITH
COMMUNITY ADVISORY BOARDS, IF
YOU HEARD ABOUT THAT, I THINK
THAT IS IMPORTANT TO KEEP A
FINGER ON THAT PULSE AS WELL.
>> YES, THANK YOU, SERGIO.
TRUST AND TRUSTWORTHINESS, I SEE
THOSE AS TWO DIFFERENT BUT
INTERTWINED TERMS.
BOTH WERE I THINK CLEAR IN MANY
OF THE ROUNDTABLE
COMMUNICATIONS, ESPECIALLY WITH
THE COMMUNITIES.
YOU KNOW, I THINK THAT IN TERMS
OF -- SO THAT'S WHY I THINK
WE'RE TRYING TO MAKE SURE THAT
IT IS FRONT AND CENTER.
I WILL GIVE YOU A LITTLE BIT OF
A GLIMPSE.
I TRY NOT TO THINK TOO FAR IN
ADVANCE.
IF YOU'RE A SPORTS TEAM YOU
DON'T WANT TO THINK ABOUT THE
TEAM YOU'RE GOING TO PLAY NEXT
WEEK.
YOU WANT TO THINK ABOUT THE ONE
TODAY.
BUT IN MAY, DURING THAT COUNCIL,
I DO PLAN ON GIVING A LITTLE BIT
MORE AROUND WHAT NCATS
SPECIFICALLY IS DOING, GIVING A
LITTLE BIT OF DEEPER DIVE, NOT
JUST IN TERMS OF THE WORK THAT
WE DO IN DATA SCIENCE BUT IN
THINKING ABOUT WHAT -- FROM THE
DATA SCIENCE PERSPECTIVE, IT'S
USING A.I.-ENABLED TOOLS AND
LARGE LANGUAGE MODELS, WE WANT
TO BE VERY CAREFUL WE'RE NOT
USING AND/OR PERPETUATING BIASED
DATA.
AND HOW CAN WE IDENTIFY WHERE
THAT MIGHT EXIST, THAT, YOU
KNOW, THAT MAY NOT BE SO EASY.
SO THINKING ABOUT WAYS IN WHICH
TO IDENTIFY AND ADDRESS THOSE
AREAS OF BIAS WILL BE ONE THING.
BUT THE OTHER THING IS TO, YOU
KNOW, WHAT THE WORK THAT HAS
BEEN DONE THROUGH THE LITCOIN
MODELS AND LLM IDEAS, THINKING
ABOUT REALLY ENGAGING THE
COMMUNITY TO SAY, YOU KNOW,
WE'VE DEVELOPED THESE
ALGORITHMS, THESE A.I.-ENABLED
ALGORITHMS, BUT WE NEED TO KNOW
FOR SURE THAT THEY ARE PRODUCING
WHAT WE EXPECT TO SEE.
AND SO ENGAGING THE COMMUNITY TO
HELP US, YOU KNOW, KICK THE
TIRES, SO TO SPEAK, ON THAT I
THINK WILL ALSO ADD TO THAT
TRUST AND TRUSTWORTHINESS OF THE
DATA.
BUT ALSO JUST AS A PRELUDE TO
WHAT I HOPE TO TALK ABOUT MORE
IN MAY IS SOME OF THE WORK THAT
PRE-CLINICAL MODELS THAT IES-
TALKED ABOUT, THE NEW APPROACH
METHODOLOGIES.
IT IS DATA IN SOME RESPECTS, BUT
IT'S ALSO THE ACTUAL CELLS AND
THE METHODS AND APPROACHES TO DO
MORE PREDICTIVE MODELING.
THERE AGAIN, YOU KNOW, WE'VE
SEEN IT OVER AND OVER AGAIN IN
THE LITERATURE THERE'S BIAS IN,
YOU KNOW, PERHAPS WHERE YOU GET
THE CELL TYPES, WHO YOU GET
THOSE FROM, EVEN IN CLINICAL
TRIALS WE KNOW THERE ARE GROUPS
THAT ARE UNDERREPRESENTED IN
CLINICAL TRIALS.
AND SO WE WANT TO MAKE SURE THAT
EVEN IN THE PRE-CLINICAL SPACE,
THE WORK THAT'S BEING DONE AT
THE CELLULAR LEVEL, THAT WE'RE
PAYING ATTENTION TO THOSE
FACTORS AS WELL.
SEX AS A BIOLOGICAL VARIABLE AND
THE ORIGINATION OF THE CELLS AND
SOURCES SO WE CAN MAKE SURE THAT
WE'RE BEING DIVERSE, INCLUSIVE
IN THOSE ACTIVITIES.
MORE SPECIFICALLY, EVALUATING
THAT AS A WAY IN WHICH WE CAN
UNDERSTAND THE EFFECTIVENESS OF
THAT MODEL IN AND OF ITSELF.
SO WE'RE TRYING TO MAKE THAT
INCLUSIVENESS, THAT DIVERSITY,
THAT TRUSTWORTHINESS AND INFUSE
THAT INTO MODELS WE'RE
DEVELOPING IN EARLIER PHASES SO
THEY CAN BE MORE SORT OF
INGRAINED AS WE GO FORWARD IN
THE DEVELOPMENT PROCESS.
>> THANK YOU.
>> ANIKA?
>> THANK YOU.
AGAIN, VERY IMPRESSIVE.
I ECHO SERGIO'S COMMENT.
YOU STARTED TO TOUCH ON ONE OF
THE COMMENTS I HAD, AS YOU'RE
BUILDING UP SO MUCH THE LINKAGES
BETWEEN WHAT YOU'RE DOING, AS
YOU SPOKE TO I THINK IT WOULD BE
REALLY INTERESTING.
SO, YOU KNOW, LIKE THE
PRE-CLINICAL RESEARCH YOU'RE
DOING, HOW LITCOIN -- HOW THAT
CAN BE LEVERAGED IN LITCOIN, AS
YOU MENTIONED.
THE SAME AS YOU'RE MOVING INTO
PHASE 2 WITH GENE EDITING,
MODELS FOR RARE DISEASE WITH
FACTOR IN, GLAD TO SEE THOSE
LINKAGES ARE STARTING TO BE
THOUGHT ABOUT.
THE OTHER COMMENT, TWO MORE
COMMENTS, I HAD, ONE WITH THE
TRANSLATIONAL SCIENCE PRINCIPLE
STICKERS, I'M WONDERING HOW WE
CAN GET THOSE OUT AS MUCH AS
POSSIBLE.
YOU KNOW, IT WOULD BE GREAT TO
SEE THAT LIKE EVEN IN PRIVATE
SECTOR INDUSTRIES, RIGHT?
BECAUSE THEY ARE STARTING TO
BUILD TRANSLATIONAL SCIENCE
TEAMS.
THESE ARE PRINCIPLES THEY MAY
PROMOTE FOR THEIR TEAMS.
SO I WOULD LOVE TO SEE HOW TO
DISSEMINATE THOSE STICKERS FAR
AND WIDE.
>> THAT'S A GREAT IDEA.
WE HAVEN'T THOUGHT ABOUT, YOU
KNOW, SORT OF SECONDHAND
DISSEMINATION BEYOND CTSAs AND
RARE DISEASE GROUPS BUT IT'S A
GREAT, AND I'LL START THINKING
ABOUT THAT.
>> AND COMMUNICATION, THE IND
SUBMISSIONS, THAT'S FANTASTIC,
WONDERING HOW THAT STORY IS
BEING TOLD AS WELL.
AND THAT DOVETAILS INTO MY LAST
COMMENT OF WITH THE STRATEGIC
PLAN AS YOU'RE DRAFTING IT, I
LOOKED AT THE OLD ONE AND DIDN'T
SEE LIKE AN IMPACT SECTION.
AND PERHAPS AT THE TIME IT MAY
BE TOO EARLY BUT I THINK TRYING
TO BUILD ON GETTING THE MESSAGE
OUT OF NCATS IMPACT AND THINKING
ABOUT WHO WILL BE SEEING THE
STRATEGIC PLAN I THINK HAVING
IT, YOU KNOW, CAPTURED IN THAT
STRATEGIC PLAN LIKE A GRAPHIC
OR, YOU KNOW, EVEN WRITTEN
SECTION THAT TALKS ABOUT THE BIG
WINS OF NCATS COULD BE HELP
FOLLOW HAVE AT THE BEGINNING OF
THE STRATEGIC PLAN.
>> ANIKA, YOU'RE READING OUR
MINDS FOR SURE.
ONE OF THE THINGS WE'VE DONE, TO
THAT END, ON NEWLY ROLLED OUT
WEBSITE WE HAVE AN IMPACT
SECTION EXACTLY FOR THAT REASON.
I THINK IT'S STILL NEW.
WE'RE EXPLORING HOW BEST TO USE
THAT.
BUT MEREDITH AND I TALKED ABOUT
THIS WITH TEAMS AS WELL, IMPACT
IS SO IMPORTANT FOR US AS WELL.
WE DON'T WANT TO HAVE A
STRATEGIC PLAN THAT'S WRITTEN
AND DONE.
WE WANT A STRATEGIC PLAN THAT'S
MEASURABLE, AND SHOWS THAT
IMPACT.
I REALLY RESONATE WITH YOUR
IDEAS HERE, AND WE DO PLAN TO
HAVE WAYS IN WHICH WE CAN TALK
ABOUT THAT MORE.
I WANT TO JUST TAKE A MOMENT TO
PAUSE BECAUSE I DO SEE DR.
BERTAGNOLLI ON THE SCREEN.
HELLO.
>> HELLO.
>> AND I WOULD LIKE TO JUST
OFFER THE IDEA OF -- MONICA, ARE
YOU READY TO GO?
WOULD YOU LIKE TO PROCEED?
>> WHATEVER YOU LIKE.
YOU CAN CERTAINLY GO FINISH WITH
THIS CONVERSATION IF YOU LIKE.
I DON'T NEED TO INTERRUPT IT.
>> OKAY.
WE'LL TAKE ONE MORE QUESTION AND
WE WILL TURN IT OVER TO YOU AND
PICK UP ON THE BACK END BEFORE
THE BREAK IF THAT SOUNDS OKAY.
SO, MARSHALL, PLEASE GO AHEAD.
>> SO, LIKE I SAID, GREAT
OVERVIEW.
ONE OF THE THINGS THAT WOULD BE
REALLY FASCINATING, SOMETHING
PAUL HARRIS AND I HAVE BEEN
BOUNCING OFF, HOW DO WE USE SOME
MACHINE LEARNING SYSTEMS,
CHATBOT TYPE SYSTEMS WHEN WE'RE
STARTING TO INCORPORATE THIS, A
LOT OF THESE NATURAL HISTORY
STUDIES WE'RE WORKING ON.
BECAUSE RIGHT KNOW WE
STRAITJACKET OURSELVES WITH
FORMALISTIC QUESTIONS, MISSING
INFORMATION ABOUT WHAT WE DON'T
KNOW WE DON'T KNOW.
ONE THING I'D LOVE TO SEE IN
THAT STRATEGIC PLAN IS -- AND
IT'S NOT -- YOU KNOW, IT'S NOT A
PANACEA FOR EVERYTHING BUT
THERE'S WAYS THOSE THINGS CAN
INTERACT WITH PATIENTS GIVING
INFORMATION THAT WILL BE MORE
EFFECTIVE AND PROBABLY ALLOW FOR
BROADER DATA COLLECTION ACROSS
SOME OFS THEY THINGS SO I'D LOVE
TO SEE THAT BUILT IN THERE.
>> A GREAT IDEA.
A GOOD USE OF THAT AS A TOOL.
YOU KNOW, IT COULD ALSO BE A WAY
IN WHICH -- YOU KNOW, IF YOU
LOOK EVEN BEYOND USING
A.I.-ENABLED OR MACHINE LEARNING
AND ALGORITHMS EVEN WITH MORE
CUTTING EDGE TOOLS YOU CAN START
TO FILL IN SOME BLANKS YOU MAY
NOT SEE IN NATURAL HISTORY
STUDIES.
>> RIGHT.
THAT'S WHAT I MEAN, WAYS TO PULL
THE DATA TOGETHER THAT SIMPLY
YOU CAN'T DO BECAUSE PEOPLE
DON'T HAVE THE TIME OR THE
BREADTH FOR THAT.
I THINK THAT WOULD BE INCREDIBLY
POWERFUL.
>> COMPLEX OF THOSE DATA ARE
PRETTY BIG SO HAVING THE MACHINE
HELP WITH THAT IS GOOD, AND THEN
HAVING THE HUMANS TEST IT.
SOUNDS LIKE AN IDEAL THING WE
CAN CONSIDER, MAYBE WHEN WE MEET
WITH THE FDA IN MAY THAT MIGHT
BE ONE THING WE CAN TALK A
LITTLE BIT ABOUT IN THINKING
MORE --
>> THINKING ALONG THOSE LINES,
PARTICULARLY POST-MARKETING
STUDIES.
>> YEAH.
>> WHAT YOU DON'T KNOW IS WHAT
WILL ACTUALLY BITE YOU.
>> EXACTLY.
WE'RE TALKING ABOUT THE KIND OF
THINGS WE'RE THINKING.
AND WE WANT TO MAKE SURE THAT
WE'RE SETTING THE STAGE SO THAT
OUR COMMUNITIES CAN NAVIGATE
THAT AS WELL.
SO, I LOVE THE IDEA, MARSHALL.
LET'S KEEP TALKING.
>> ALWAYS.
>> ALL RIGHT.
WITH THAT, I THINK WE'RE
PROBABLY A LITTLE BIT EARLY.
GREG, ARE WE OKAY TO GO AHEAD
AND MOVE FORWARD WITH DR.
BERTAGNOLLI?
EVERYTHING OKAY ON YOUR END?
>> YEP.
THAT'S PERFECTLY FINE.
>> ALL RIGHT.
WELL, I'M SUPER, SUPER, SUPER,
EXCITEMENTED TO HAVE THE 17th
DIRECTOR OF THE NIH HERE TODAY,
DR. MONICA BERTAGNOLLI.
SHE'S THE FIRST SURGEON AND THE
FIRST -- ACTUALLY THE SECOND
WOMAN TO HOLD THE POSITION.
NOMINATED BY PRESIDENT BIDEN ON
MAY 15 OF 2023, CONFIRMED ON
NOVEMBER 7, TOOK OFFICE ON
NOVEMBER 9.
AND PREVIOUSLY JUST BEFORE THAT
SHE SERVED AS 16th DIRECTOR OF
NCI, AND SHE WAS RICHARD WILSON
PROFESSOR OF SURGERY AT HARVARD,
YOU ARE SURGEON AT BRIGHAM AND
WOMEN'S, AND DANA-FARBER CANCER
INSTITUTE.
DR. BERTAGNOLLI HAS BEEN AT THE
FOREFRONT OF CLINICAL ONCOLOGY.
HER LAB FOCUSED ON ADVANCING
UNDERSTANDING OF THE GENETIC
DRIVERS OF GASTROINTESTINAL
CANCER AND ROLE OF INFLAMMATION
AS PROMOTER OF CANCER GROWTH.
AND THEN AS A PHYSICIAN AND
SCIENTIST SHE LED TRANSLATIONAL
SCIENCE INITIATIVES WITHIN THE
NCI-FUNDED COOPERATIVE GROUPS
PROGRAM, NOW KNOWN AS NCI'S
NATIONAL CLINICAL TRIALS
NETWORK.
AND THEN SHE ALSO WAS CHAIR OF
THE ALLIANCE FOR CLINICAL TRIALS
IN ONCOLOGY, NATIONAL CLINICAL
TRIALS NETWORK MEMBER
ORGANIZATION, SHE HAS HUGE
FAMILIARITY OF COURSE WITH
CLINICAL TRIALS.
SHE HAS CHAMPIONED A VARIETY OF
COLLABORATIVE INITIATIVES TO
TRANSFORM DATA INFRASTRUCTURE
FOR CLINICAL RESEARCH AND IS THE
FOUNDING CHAIR OF THE MINIMAL
COMMON ONCOLOGY DATA ELEMENTS,
N-CODE, ALSO PAST PRESIDENT AND
CHAIR BOARD OF DIRECTORS
AMERICAN SOCIETY OF CLINICAL
ONCOLOGY, AND JUST A LITTLE
TID-BIT IS THAT SHE IS THE
DAUGHTER OF A FIRST GENERATION
ITALIAN, FRENCH BASQU AS
IMMIGRANTS, GREW UP ON A RANCH
IN SOUTHWESTERN WYOMING.
WE SHARE A LOVE FOR WEARING
COWBOY BOOTS.
GRADUATED FROM PRINCETON,
MEDICAL SCHOOL UNIVERSITY OF HUE
THAT, TRAINED AT BRIGHAM AND
WOMEN'S, FELLOWSHIP AT
DANA-FARBER.
GREAT TO HAVE YOU.
THANK YOU FOR JOINING US.
>> THANK YOU, JONI.
I'M SO THRILLED TO BE ABLE TO BE
WITH YOU ALL TODAY!
AND I SERVED IN THE SISTER
ORGANIZATION TO NCATS, I'VE
SERVED IN THE NCTN, BUT I'M
REALLY ACTUALLY QUITE FAMILIAR
WITH YOUR WORK, HAVING BEEN,
LIKE I SAID, NEARBY COLLABORATOR
OVER MANY YEARS.
SO DELIGHTED TO BE HERE TODAY TO
MEET WITH YOU.
OKAY.
LET'S SEE IF I CAN MAKE MY
SLIDES WORK.
OH, OKAY.
JONI ALREADY SAID THIS IN THE
INTRODUCTION, HERE'S WHERE I
GREW UP.
IT REALLY IS IN THE MIDDLE OF
NOWHERE.
SO I AM A RURAL PERSON.
I GREW UP IN A SPOT, THIS IS
WHAT IT IS.
THE FIRST NEIGHBOR, THE NEXT
HOUSE, WAS ABOUT 8 MILES AWAY.
THE NEXT PAVED ROAD WAS 18 MILES
OF DIRT ROAD BEFORE YOU HIT THE
PAVEMENT.
SO WE REALLY WERE IN A VERY
REMOTE LOCATION.
BUT YOU ALSO SEE WHAT I'M
INCREDIBLY PROUD OF, THAT I'M
CURRENTLY CREDENTIALED TO BE A
PHYSICIAN IN THE CLINICAL CENTER
AT THE NIH, HERE IS ME DOWN IN
THE BOTTOM RIGHT.
SERVING AS FIRST ASSISTANT TO A
FORMER POSTDOC IN MY LAB,
SURGICAL ONCOLOGY TRAINEE NOW
ONE OF THE SENIOR SURGEONS AT
THE NIH CLINICAL CENTER IN THE
IMMUNOTHERAPY BRANCH, DR.
STEPHANIE GOFF.
THERE'S NOTHING MORE PRECIOUS IN
YOUR CAREER THAN WHEN YOU GET TO
COME IN AND BE THE FIRST
ASSISTANT OF A FORMER TRAINEE.
AND IT'S REALLY VERY THRILLING.
SO TODAY'S DISCUSSION WE'RE
GOING TO TALK ABOUT WHAT WE'RE
TRYING TO DO TO GET NIH REALLY
ENERGIZED, IN AN OPPORTUNITY
THAT A NEW DIRECTOR HAS TO DO
SO.
SOME THINGS THAT I THINK ABOUT
AS GUIDING PRINCIPLES A LITTLE
BIT ABOUT OUR BUDGET, AND A
LITTLE BIT ABOUT VISION.
SO FIRST, THIS IS SOMETHING WE
ALL HAVE TO DEAL WITH, THE
HEALTH OF THE U.S. POPULATION IS
DECLINING.
WE HAVE TO REVERSE THIS TREND.
WE'RE GETTING A VERY BAD REPORT
CARD RIGHT NOW.
THERE'S MUCH THAT CAN DIVIDE US
RIGHT NOW.
HOWEVER, IT'S VERY CLEAR THAT
THE DESIRE FOR LONG HEALTHY
LIVES FOR OURSELVES AND OUR
LOVED ONES IS A UNIFYING AND
GALVANIZING FORCE ACROSS ALL
DISCIPLINES, ALL WALKS OF LIFE,
ALL POLITICAL ARENAS.
SO WE NEED TO REALLY EMBRACE
THAT.
BASED ON MY DISCUSSIONS WITH
MEMBERS OF CONGRESS, I KNOW ALL
OF THEM CARE VERY DEEPLY ABOUT
THIS ISSUE FOR THEIR
CONSTITUENTS.
AND THE OTHER THING THAT'S
CRITICAL, WE HAVE TO APPROACH
OUR WORK WITH GREAT URGENCY,
BECAUSE WE'RE NOT WINNING RIGHT
NOW.
AS YOU MAY KNOW, U.S. LIFE
EXPECTANCY IS LOW COMPARED TO
OUR PEER NATIONS.
THIS IS NOT A PANDEMIC AREA
ISSUE.
LIFE EXPECTANCY WAS DECLINING IN
THE U.S. WELL BEFORE THE
PANDEMIC.
AND DESPITE SEEING INCREASE IN
2022, WE HAVE STILL NOT MADE UP
FOR ADDITIONAL LOSSES DURING THE
PANDEMIC AND WE'RE NOT ON THE
RIGHT TRACK.
THERE'S THIS UGLY FACT.
EVEN THOUGH WE'RE NOT DOING AS
WELL AS COMPARATOR COUNTRIES,
OUR LIFE EXPECTANCY IS VERY LOW
COMPARED WITH PEER COUNTRIES,
EVEN THOUGH WE SPEND ENORMOUSLY
MORE ON OUR HEALTH CARE SYSTEM.
AND THIS IS DRILLING DOWN TO
REALLY UNDERSTAND WHERE AND WHY
AND IS REALLY INCREDIBLY
SOBERING.
WE ACCORDING TO A 2021 REPORT
FROM NATIONAL ACADEMIES, THIS
DECLINE IN LIFE EXPECTANCY WE'RE
SEEING SEEMS LARGELY RELATED TO
AN INCREASING MORTALITY AMONG
WORKING-AGE ADULTS, AGE 18-64,
AND THE MAIN DRIVERS OF THIS,
ENOUGH TO DRIVE LIFE EXPECTANCY,
ARE DRAMATIC INCREASE IN DEATHS
DUE TO DRUG POISONING, ALCOHOL
POISONING, ALCOHOL-RELATED
CAUSES, AND SUICIDE.
ALSO IMPORTANTLY, A SLOWING OF
OUR PROGRESS IN REDUCING DEATHS
DUE TO CARDIOVASCULAR DISEASE.
SO THESE ARE SOME CORE ISSUES IN
HEALTH THAT ALL OF US CAN MAKE,
CAN AND HAVE TO MAKE AN
IMPORTANT CONTRIBUTION TO
REVERSING.
THE REPORT, THESE REPORT AUTHORS
DESCRIBE A NATIONAL POPULATION
HEALTH CRISIS, A TREND THREATENS
THE FUTURE OF FAMILIES, THE
SOCIAL FABRIC OF COMMUNITIES AS
WELL AS THE PRODUCTIVITY AND
COMPETITIVENESS OF OUR COUNTRY.
SO, GUIDING PRINCIPLES, WHAT CAN
NIH DO TO ADDRESS THIS CRISIS,
ESPECIALLY IN THE TIME -- IN
CHALLENGING TIMES, AND IN TIME
OF FLAT BUDGET.
I'M STILL SHAPING PLANS AS I
MEET WITH LEADERS ACROSS THE
AGENCY AND INSTITUTE BUT I
WANTED TO SHARE SOME PRINCIPLES
THAT ARE GUIDING OUR MISSION.
FIRST, AND FOREMOST, OUR WORK IS
NOT FINISHED WHEN WE DELIVER
SCIENTIFIC DISCOVERIES.
WE'RE NOT THE NATIONAL
INSTITUTES OF DISEASE.
OUR WORK IS FINISHED WHEN ALL
PEOPLE ARE LIVING LONG AND
HEALTHY LIVES.
WE CANNOT OVEREMPHASIZE THIS.
WE HAVE TO DO EVERYTHING IT
TAKES TO ACHIEVE THIS ULTIMATE
AIM, EVEN THOUGH WE KNOW THIS
ULTIMATE AIM WON'T HAPPEN
WITHOUT ROBUST, SUSTAINED
ATTENTION TO ADVANCING
SCIENTIFIC DISCOVERIES.
HERE'S THE OTHER ONE, NOT TO
FORGET THE NIH RESEARCH
ENCOMPASSES, ABSOLUTELY
DEPENDENT UPON, THE LABORATORY,
THE CLINIC, AND THE COMMUNITY.
I THINK WE'VE KNOWN ABOUT THE
LABORATORY AND CLINIC AND TALKED
ABOUT THAT FOR A LONG TIME, BUT
INCREASINGLY WE KNOW THAT WE
CAN'T ACHIEVE OUR AIMS WITHOUT
ALSO CONSIDERING THE COMMUNITY
TO BE OUR ARENA FOR RESEARCH.
WE HAVE AN UNPRECEDENTED
OPPORTUNITY TO EMBRACE AND
INCREASE ACCESS TO INNOVATION.
I REALLY WANT TO FOCUS ON WHAT
NIH CAN DO AS A BIOMEDICAL
RESEARCH INSTITUTION TO ENSURE
OUR ADVANCES REACH THE PEOPLE
WHO NEED THEM, AND FOR ME THIS
IS PERSONAL BECAUSE AS I SHOWED
YOU, I GREW UP IN A VERY RURAL
AREA, ONE OF THE SEGMENTS OF OUR
SOCIETY WHICH HAS
DISPROPORTIONATELY BAD HEALTH
OUTCOMES.
SO, PROGRESS IS ACCELERATED WHEN
ADVANCED SCIENTIFIC METHODS SUCH
AS NEW DATA ANALYTICS ARE
APPLIED TO DATA THAT INCLUDE
EVERYONE.
WHEN NEW DISCOVERIES ARE RAPIDLY
AND EQUITABLE ADOPTED IN
CLINICAL CARE.
I CAN'T IMAGINE THIS SPEAKS TO
ANY PART OF NIH MORE THAN IT
DOES TO YOU ALL AT NCATS.
NEW ADVANCES IN INFORMATION AND
TECHNOLOGY AND DATA SCIENCE,
WHILE FAMILIAR TO RESEARCHERS,
HAVE CAPTURED VERY SIGNIFICANT
PUBLIC ATTENTION NOW THAT, YOU
KNOW, WE HAVE THE ARRIVAL OF
LARGE LANGUAGE MODELS, ANYONE
CAN USE CHATGPT, THE WORLD IS
TALKING BUT WE'VE BEEN THE
DEVELOPERS OF THESE NEW METHODS,
OBVIOUSLY.
BUT NOW IT'S GOT EVERYBODY'S
ATTENTION.
SO THIS PROVIDES A GREAT
OPPORTUNITY FOR US ACTUALLY.
OUR KNOWLEDGE AND TECHNOLOGY
HAVE DEVELOPED TO THE POINT
WHERE WE SHOULD BE ABLE TO
DELIVER EVIDENCE-BASED
DATA-DRIVEN HEALTH CARE TO EVERY
PATIENT.
YOU ALL KNOW WE ARE NOT DOING
THAT TODAY.
BUT THE TECHNOLOGY IS THERE TO
DO IT.
SO, THIS IS NOT A TECHNOLOGY
PROBLEM.
THIS IS A SOCIAL ENGINEERING
PROBLEM.
AND, AGAIN, I AM SO DELIGHTED TO
BE ABLE TO TALK TO THIS GROUP
BECAUSE YOU ARE ALL ON THE
FOREFRONT OF BEING ABLE TO SOLVE
THIS.
ARTIFICIAL INTELLIGENCE IS
ALREADY REVOLUTIONIZING WHAT IS
POSSIBLE IN MEDICAL RESEARCH AND
CARE, BUT TO REALIZE ITS
POTENTIAL FOR IMPROVING HEALTH,
WE ABSOLUTELY HAVE TO INVEST IN
A SECURE AND SUSTAINABLE DATA
SHARING INFRASTRUCTURE THAT
REACHES EVERYONE.
SO, THE BUDGET CLIMATE, ALL OF
THESE REALLY CRITICAL IMPORTANT
NEEDS REACHING BEYOND WHERE WE
ARE TODAY ARE SET IN A TIME OF
ALSO VERY CHALLENGING FISCAL
RESTRAINTS.
AS I NOTED EARLIER I WANT TO
FOCUS ON WHAT NIH CAN DO AS A
BIOMEDICAL RESEARCH INSTITUTE TO
ENSURE THAT OUR ADVANCES REACH
THE PEOPLE WHO NEED THEM.
BUT RIGHT NOW WE'RE ALL VERY
CONCERNED ABOUT THE BUDGETARY
PRESSURES WE WILL FACE AND
DIFFICULT DECISIONS WE WILL NEED
TO MAKE IN THE NEAR TERM.
I'M HOPEFUL CONDITIONING WILL
AVOID DRASTIC CUTS TO NIH AND
SIBLING AGENCIES ACROSS HHS BUT
A FLAT BUDGET WHICH WE'RE
EXPECTING WILL HAVE A
SIGNIFICANT NEGATIVE IMPACT.
WE ALWAYS SEEK OUT THE BEST NEW
IDEAS, AND BEST NEW SCIENCE, AND
EVEN WITH FUNDING WE CURRENTLY
RECEIVE WE'RE LEAVING GREAT
IDEAS AND POTENTIAL FOR NEW
DISCOVERIES ON THE TABLE.
IN TIMES OF STRESS, SUCH AS
THIS, IT IS THE YOUNGER
GENERATION OF RESEARCHERS THAT
ARE ALWAYS THE MOST ADVERSELY
AFFECTED.
WE COULD LOSE A GENERATION OF
RESEARCHERS IF WE DON'T DO
EVERYTHING WE CAN TO SUPPORT
THEM DURING THIS TIME.
WE NEED TO DO EVERYTHING WE CAN
TO CONTINUE TO FUND THE BEST
SCIENCE, AND TO SUPPORT THOSE
WHO WILL TAKE OVER WHEN WE ARE
DONE.
SO, WHAT SHOULD NIH DO?
ONE OF THE THINGS I LOVE ABOUT
BEING AT NIH IS BEING AMONG SO
MANY THOUGHTFUL AND DEDICATED
PEOPLE WHO ARE FOCUSED ON WHAT
NIH CAN DO TO IMPROVE HEALTH FOR
EVERYONE.
AND I'M ENJOYING DISCUSSING
IDEAS FOR WHAT NIH SHOULD DO TO
HAVE THE MOST IMPACT FOR HEALTH.
THIS IS A TIME OF VERY SERIOUS
PRIORITY SETTING EVERYTHING
STILL FLESHING OUT DETAILS WHAT
I HOPE TO PROMOTE IN MY TIME IN
LEADERSHIP AT NIH.
I HAVE TWO PRIMARY PRIORITIES
THAT I HOPE TO PUT IN PLACE AS
NIH DIRECTOR.
FIRST IS TO CORRECT RESEARCH TO
PRIMARY CARE.
MY HOPE IS WE CAN BETTER MEET
PEOPLE WHERE THEY ALREADY
RECEIVE CARE, TO BETTER ENGAGE
ALL COMMUNITIES INCLUDING
UNDERREPRESENTED ONES.
I'LL GO BACK TO MY OWN PERSONAL
EXPERIENCE.
PEOPLE IN WYOMING, EVEN AS
REMOTE AS THE PLACE WE LIVED,
COULD HAVE ACCESS TO A PRIMARY
CARE DOCTOR.
WASN'T ALWAYS EASY BUT THE FIRST
LINE OF DEFENSE WAS ALWAYS THE
PRIMARY CARE DOCTOR, SPECIALLY
CARE WAS EVEN HARDER.
WE REALLY HAVE TO FOCUS ON THIS
AT, YOU KNOW, THE FIRST LINE OF
DEFENSE, THE PRIMARY CARE
ENVIRONMENT.
WE NEED TO USE ELECTRONIC HEALTH
RECORD TO ENGAGE PEOPLE IN
RESEARCH.
WE NOW HAVE A TECHNOLOGY THAT IF
WE USE IT PROPERLY CAN TRULY
UNITE EVERYONE.
THE U.S. UTILIZATION NOW OF
EHRs IS IN EXCESS OF 98%.
AND SO WE HAVE A TOOL THAT CAN
BE A POWERFUL FORCE FOR DRIVING
EVIDENCE-BASED RESEARCH AND
KNOWLEDGE.
UNFORTUNATELY WE ARE NOT USING
IT THE WAY WE NEED TO AT ALL.
WHO IS GOING TO FIX THIS?
WELL, IT'S GOING TO BE NIH, IN
PARTNERSHIP WITH OUR SISTER
AGENCIES ACROSS HHS, AND THIS IS
ONE OF THE THINGS I AM
PARTICULARLY PASSIONATE ABOUT.
WE WANT TO INCREASE RESEARCH
CAPABILITIES AND EFFICIENCY WITH
INNOVATIVE STUDY DESIGNS THAT
ADDRESS COMMON HEALTH ISSUES,
CAN REALLY ADDRESS PREVENTION,
AND IMPLEMENTATION.
AND YOU ALL KNOW THAT THIS LAST
MILE RESEARCH CAN BE AMONG THE
MOST DIFFICULT TO DO, ESPECIALLY
THE MOST DIFFICULT TO
DISSEMINATE COMMUNITIES OF NEED.
FINALLY WE NEED TO RAPIDLY
DISSEMINATE EVIDENCE TO GUIDE
PATIENT AND PROVIDER DECISIONS.
AGAIN, WE HAVE TECHNOLOGY TOOLS
THAT CAN HELP DO THIS.
WE'RE NOT YET HARNESSING THEM AS
WE SHOULD.
AS I SAID EARLIER WE'RE IN A
PERIOD OF TIME WE HAVE ENORMOUS
ABUNDANCE OF DATA.
AND FEDERAL REGULATIONS NOW
REQUIRE NIH-FUNDED RESEARCHERS
TO SHARE THEIR DATA PUBLICLY.
IN ORDER TO MAKE THE BEST USE OF
THIS INFLUX OF DATA, I HOPE TO
MAKE THE NATIONAL LIBRARY OF
MEDICINE A REVITALIZED HUB FOR
EXPANDED BIOMEDICAL DATA,
RESEARCH DATA USE, NOT JUST DATA
SHARING, NOT JUST DATA STORAGE,
BUT FOCUS ON DATA USE THAT IS
ABLE TO INFORM NEW RESEARCH AND
IMPROVE HEALTH OUTCOMES.
MANY PEOPLE ASKED ME HOW ARE WE
GOING TO DEAL WITH THIS DATA
THAT COMES FROM EVERYWHERE,
EVERY DIFFERENT DIRECTION.
OUR FOCUS IS GOING TO BE ON THE
DATA WE NEED, THE DATA WE ARE
ACTUALLY USING TO IMPROVE
HEALTH.
AND TO GET THE RIGHT DATA IN THE
HANDS OF THE PEOPLE WHO ARE
ASKING IMPORTANT QUESTIONS.
SO MY VISION IS TO INTEGRATE
DATA FROM BASIC AND SOCIAL
SCIENCE RESEARCH, PUBLIC HEALTH
AND CLINICAL CARE, FOCUSED ON
USERS, NOT JUST MERELY STORAGE,
INCREASE CAPACITY FOR DATA
HOSTING, ENABLE LOW COST ACCESS
TO DATA USING OPEN INDUSTRY DATA
STANDARDS, SUPPORT BROAD ACCESS
TO ADVANCED ANALYTICS AND
COMPUTATIONAL POWER.
THE STORAGE ISSUE IS A
CONSIDERABLE ONE.
THE ONLY WAY TO DO IT, THE MOST
EFFECTIVE WAY TO DO IT, YET
PRESERVE THE CREATIVITY AND
AUTONOMY AND RESPONSIBILITY OF
INDIVIDUAL RESEARCH TEAMS IS TO
EMPLOY A FEDERATED ARCHITECTURE
FOR DATA USE, OUR PLAN.
FINALLY, WE REALLY NEED TO
IMPROVE, TO INCREASE OUR
EDUCATION AND WORKFORCE
DEVELOPMENT AS A PART OF THIS
ISSUE.
DO WE HAVE THE RIGHT PEOPLE WITH
THE RIGHT KNOWLEDGE WHO KNOW HOW
TO ENTER AND USE THIS NEW DATA
ENVIRONMENT?
SO, I THINK WE'RE GETTING TO THE
END HERE.
WHAT I'M PROPOSING ISN'T
NECESSARILY NEW.
IN FACT, FOR MORE THAN TEN YEARS
THE NATIONAL ACADEMY OF SCIENCE
PUT OUT A REPORT ON THE U.S.
HEALTH DISADVANTAGE, AND THERE
ARE TWO PRIMARY RECOMMENDATIONS.
FOCUSED ON DATA AND IMPROVED
ANALYTICS.
THE U.S. HEALTH DISADVANTAGE IS
ONLY INCREASED IN THE PAST
DECADE SINCE THIS REPORT CAME
OUT.
IT'S TIME.
NO MORE EXCUSES.
WE NEED TO PRESS FORWARD TRYING
TO FIND REAL SOLUTIONS TO THIS
MASSIVE ISSUE.
I THINK WITH THAT, I'D LIKE TO
SAY THANK YOU SO MUCH FOR
ALLOWING ME TO BE HERE.
JONI, DO WE HAVE TIME FOR A FEW
QUESTIONS, I HOPE?
>> WE SURE DO, ESPECIALLY IF YOU
HAVE TIME FOR A FEW QUESTIONS.
>> I'M ALL YOURS.
>> GREAT.
MARSHALL, OVER TO YOU.
>> LOVE THE VISION, FIRST UP.
I HATE THAT I'M ROTATING OFF
COUNCIL KNOW BECAUSE IT'S
GETTING REALLY INTERESTING.
I'VE WORKED IN MEDICINE FOR
AROUND 40 YEARS, THAT WORK IN
THE ELECTRONIC HEALTH RECORD
SYSTEM, I REALLY WOULD LOVE TO
SEE THAT FOCUS CHANGE BECAUSE
IT'S EVOLVED MORE AS A BILLING
SYSTEM THAN INFORMATION
COLLECTION SYSTEM WITH ELEMENTS
THERE, PAUL HARRIS AND I WORKED
TOGETHER FOR YEARS, THESE ARE
DISCUSSIONS WE'VE HAD.
PLEASE TACKLE THIS.
IF YOU CAN FIND THIS WAYS TO
TURN THIS INTO A TRUE HEALTH
DRIVER, IT'S THERE.
IT'S JUST WE'VE GOT TO USE IT
PROPERLY.
AND BASICALLY I'M SAYING GO GET
IT!
>> WE WILL.
>> THANK YOU, MARSHALL.
>> I'LL SAY, MARSHALL, IF YOU'VE
BEEN IN THIS SPACE YOU KNOW IT.
YOU KNOW WHAT'S HOLDING US BACK.
>> I KNOW EXACTLY WHAT'S HOLDING
US BACK.
>> TECHNOLOGY IS NOT THE DATA
STANDARD.
IT'S NOT ANYTHING.
IT'S THE WILLINGNESS TO WORK
TOGETHER.
>> MIGHT HAVE WAS WORKING IN
SENATE, THAT'S WHAT SHE WAS
DOING, THE RULES, SAYING IT HAS
TO BE TRANSPORTABLE AND OF
COURSE IMMEDIATELY LAWSUITS WERE
FILED TO BLOCK THAT.
AND IT WILL PROBABLY REQUIRE
LEGISLATIVE ACTION TO GET IT
DONE.
>> WELL, WE'RE WORKING OUT.
THE OTHER THING I WANT TO SAY
SINCE I HAVE THE PODIUM, AGAIN,
IS THAT WE HAVE A TRUST PROBLEM
IN THE UNITED STATES TOO THAT
WE'RE VERY, VERY AWARE OF AND
INTERESTED IN.
THE LAST THING WE WANT TO BE
DOING IS --
[ NO AUDIO ]
NO, YOU CAN'T.
ALSO BE AGE TO SAY YES, YOU CAN
RECONTACT ME IF THERE'S
SOMETHING RELEVANT TO ME, HOW
CRITICAL THAT WOULD BE FOR
PEOPLE WITH RARE DISORDERS, OH
MY GOODNESS, IF I WAS A MOM OF A
LITTLE BOY WITH MUSCULAR
DYSTROPHY WOULDN'T I WANT TO SAY
YES, PLEASE, CONTACT ME, IF YOU
KNOW SOMETHING?
THERE'S GOING TO BE SO MANY
PEOPLE OUT THERE JUST REALLY
DESPERATE FOR US TO BE ABLE TO
DO THIS FOR THEM.
WE'RE ALSO GOING TO DO THAT.
>> I'M OUT OF THE RARE DISEASE
FIELD.
WITH REGISTRIES WE SET UP WHEN
WE PUT IN THAT QUESTION, ARE
YOU OKAY WITH SHARING DATA,
WE'RE NORTH OF 90%.
AS LONG AS PEOPLE CAN OPT OUT IT
MAKES FOR A GREAT SYSTEM.
>> THE LAST MILE WE NEED TO GO,
TRULY AMBITIOUS, BUT WE NEED TO
DO IT, IS THAT LET'S SAY YOU ARE
A RARE -- YOU KNOW, ARE YOU
SOMEBODY WHO SAYS YES, PLEASE,
I'M GIVING YOU MY DATA, BOY, WE
HAVE THE ABILITY TO THEN GO BACK
TO THEM AND SAY, HEY, HERE IS A
PAPER PUBLISHED THAT INCLUDED
YOUR DATA.
THERE'S NO REASON WHY WE CAN'T
DO THIS.
WE'RE ALSO WORKING THAT IN SO
THAT PEOPLE DON'T JUST BLINDLY
GIVE IT BUT THEY CAN ACTUALLY
SEE WHAT WE'VE DONE WITH IT.
I'M EXCITED THAT THE TEAM'S
BUILDING THAT IN.
>> YEAH, THAT DASHBOARDING IS
ALWAYS IMPORTANT.
>> YEAH.
OKAY, SORRY.
THANKS.
>> THANK YOU.
>> PAUL SHIREMAN?
>> LOVED YOUR TALK.
AS A FELLOW SURGEON, I CAN'T
AGREE MORE.
I PRACTICE CLINICALLY, HAVE A
LOVE AND MOSTLY HATE
RELATIONSHIP WITH THE ELECTRONIC
HEALTH RECORD, HOW UNFRIENDLY IT
IS TO CLINICIANS, WE'VE TAKEN
THE HIGHEST PAID CLINICIANS AND
MADE THEM SECRETARIES AND DATA
ENTRY PEOPLE RATHER THAN HAVING
THEM FOCUS ON THE PATIENT.
THE DATA SHARING, THE IMPORTING,
SO I THINK ONE OF THE CHALLENGES
ESPECIALLY INTEGRATING AS A
SURGEON THE PRIMARY CARE AND
TRYING TO GET THAT WHOLE
ECOSYSTEM TO WORK IS SOME OF THE
SIMPLEST THINGS ARE ACTUALLY
GOING TO BE THE MOST EFFECTIVE.
I LOVE NIH BUT OF COURSE YOU
WANT SOMETHING SEXY, COOL, STUDY
SECTION, THAT'S WHAT THEY WANT.
HAVING THE SIMPLE THINGS WE
AREN'T DOING, MEDICATION
ADHERENCE, MORE EFFECTIVE
BILLING CODES SO PRIMARY CARE
DOCS WITH CALL AND MAKE SURE
PATIENTS ARE TAKING MEDICATIONS,
USING A.I., THAT'S JUST ONE
EXAMPLE.
SO, I REALLY WOULD BE FASCINATED
ON YOUR THOUGHTS ABOUT HOW WE
TURN THIS SHIP AND IMPROVE OUR
DISSEMINATION AND IMPLEMENTATION
RESEARCH SO WE GET THINGS OUT
MORE AND AS A GIRL WHO GREW UP
ON A FARM TOTALLY GET THE WHOLE
RURAL THING, HAVING TO BE
PATIENT NAVIGATOR FOR MY RURAL
FAMILY THAT STILL LIVES IN
INDIANA.
>> YEAH, WELL, THAT'S GREAT.
YEAH, OKAY.
SO, WE HAVE TWO -- THERE'S TWO
GROUPS WE'RE HERE TO SERVE.
WE ARE HERE TO SERVE THE
PATIENTS IDEALLY, PEOPLE EVEN
BEFORE THEY ARE PATIENTS, AND
THE CLINICIANS.
THE CLINICIANS AND CAREGIVERS.
AND, YOU KNOW, SOMETIMES
CAREGIVER IS EVEN FAMILY.
THOSE ARE THE FRONT LINE PEOPLE
WE'RE HERE TO SERVE.
AND ANYTHING WE PRODUCE HAS
TO -- THAT'S WHERE IT ALL
HAPPENS.
IT DOES NOT HAPPEN ANYWHERE ELSE
BUT AT THAT CLINICIAN/PATIENT
RELATIONSHIP, WHERE EVERYTHING
COMES DOWN TO.
SO EVERYTHING WE DO HAS TO
ENHANCE THAT AND FACILITATE
THAT.
THE OTHER THING I WILL SAY THAT
NIH IS THE RESEARCH ARM.
WE TEST WHAT WORKS, FIGURE OUT
WHAT WORKS AND DOESN'T WORK.
I'M ALSO THRILLED AT THE
ENVIRONMENT WE HAVE RIGHT NOW
ACROSS HHS, I MEET EVERY SINGLE
WEEK WITH LEADERS IN CMS, CDC,
FDA, IN FACT I'M TALKING TO TWO
FDA PEOPLE THIS WEEKEND.
YOU KNOW, ABOUT HOW DO THESE
AGENCIES ALSO WORK TOGETHER TO
DEVELOP DECISION BECAUSE WE CAN
DO THE RESEARCH BUT WE'RE NOT
CARE DELIVERY.
WE'RE NOT THE CARE DELIVERY
INSTITUTE.
HOW THAT ALL WORKS TOGETHER IS
REALLY CRITICAL AND THE
ENVIRONMENT RIGHT NOW IS VERY
FAVORABLE FOR THAT.
IT'S, YOU KNOW, IT COMES AND
GOES WITH ADMINISTRATIONS.
THIS IS ANOTHER REASON WHY I
CONSIDER WHAT WE'RE DOING RIGHT
NOW TO BE INCREDIBLY URGENT.
BECAUSE WE'VE GOT THE TEAM RIGHT
NOW THAT REALLY WANTS TO DO
THIS.
SO WE BETTER RUN AS FAST AS WE
CAN.
>> GREAT.
THANK YOU.
SERGIO?
>> YES, THANK YOU, JONI.
I WILL ADD MY WARM WELCOME TO
YOU, DR. BERTAGNOLLI.
AND THANK YOU, JONI, FOR MAKING
THIS HAPPEN.
MANY THINGS THAT YOU MENTIONED
ARE MUSIC TO MY EARS.
AND BECAUSE WHAT IS BEHIND OR
UNDERLYING THE DATA THAT YOU
PRESENTED ON -- THAT WE'RE NOT
DOING WELL IN TERMS OF, YOU
KNOW, LIFE EXPECTANCY, ARE
HEALTH INEQUITIES.
AND I HAVE READ THAT IS ONE OF
YOUR AREAS OF -- THAT YOU'RE A
CHAMPION IN THAT, YOU'RE A
CHAMPION IN ALSO, THE WORK WITH
UNDERSERVED COMMUNITIES.
AND I WAS PARTICULARLY DELIGHTED
IN ONE OF YOUR SLIDES THAT YOU
PRESENTED ABOUT WHAT NIH COULD
DO, OR SHOULD DO, THAT YOU
MENTIONED MEET PEOPLE WHERE THEY
ALREADY RECEIVE CARE THROUGH
BETTER ENGAGEMENT,
UNDERREPRESENTED COMMUNITIES,
THAT'S ABSOLUTELY MUSIC TO OUR
EARS.
YOU KNOW, THAT'S WHERE THINGS
ARE HAPPENING.
AND IF YOU ALLOW ME TO PUSH A
LITTLE BIT FURTHER, THAT
FOCUSING ON THOSE WHO ARE COMING
TO RECEIVE SERVICES, IT'S
IMPORTANT, BUT FOR DECADES WE
KNOW WHAT ARE THE BARRIERS OF
ACCESS TO CARE, OF MANY
POPULATIONS, AND ALSO RELATIVELY
LACK OF STABILIZATION, I WOULD
LOVE FOR YOU TO COMMENT, YOU
KNOW, IF WE CAN PUSH A LITTLE
BIT FURTHER THAT INSTEAD OF
BEING ON THE WAITING MODE, YOU
KNOW, COME TO US, SO WE CAN TAKE
CARE OF YOU, THAT WE WORK WITH
SYSTEMS THAT WE GO WHERE PEOPLE
ARE AT AND OVERCOME THOSE
BARRIERS OF ACCESS TO CARE, IT
WOULD BE FANTASTIC AND I THINK
IT WOULD GET I ANTICIPATE TO
THOSE TRUNCATED LIFE
EXPECTANCIES THAT WE SAW SO, YOU
KNOW, THIS IMPACT DURING THE
PANDEMIC ESPECIALLY.
AND ONE LAST COMMENT, I WAS
DELIGHTED THAT JONI TALKED ABOUT
YOUR BACKGROUND, ABOUT BEING
ITALIAN BASQUE.
I'M DELIGHTED, MY
[SPEAKING IN A FOREIGN LANGUAGE]
IS BASQUE IS WELL.
>> MY MOTHER'S LAST NAME IS
KATAKOBURO.
>> CANNOT BE MORE BASQUE.
>> A QUICK STORY.
I'VE BEEN SO FORTUNATE TO HAVE
WORKED WITH SOME AMAZING PEOPLE.
SO, I HAVE A VERY GOOD FRIEND,
ONE FRIEND WHO HER NAME IS
PEGGY, SHE'S A SURGEON, WORKS IN
QUEENS, NEW YORK.
SHE'S THE DIRECTOR OF THE QUEENS
CANCER CENTER.
DO YOU KNOW WHAT PEGGY DID?
PEGGY-- QUEENS IS CRAZY, YOU
KNOW, THREE-QUARTERS OF PATIENTS
ARE ON MEDICAID, IT'S NEW YORK.
SO GET MEDICAID OR SIGNED UP,
THEY DON'T HAVE INSURANCE, MANY
ARE BRAND-NEW IMMIGRANTS, MANY,
YOU KNOW, WHAT DID PEGGY DO?
IN HER CLINIC THERE'S A FOOD
PANTRY.
IN HER CLINIC THERE'S ALSO A DAY
CARE.
BECAUSE THE MOTHER WHO NEEDS TO
COME IN TO HAVE HER CHEMOTHERAPY
DOESN'T HAVE ANYONE TO WATCH THE
CHILDREN.
YOU KNOW, SO PEGGY HAS ALWAYS
TAKEN THE APPROACH, AND SHE USED
TO TELL ME, IT'S A BEAUTIFUL
EXPRESSION, WHAT IF YOUR BIGGEST
PROBLEM IS NOT THAT YOU HAVE
CANCER, YOU HAVE BIGGER PROBLEMS
THAN THAT?
YOU KNOW.
IT'S JUST -- THESE KINDS OF
SOLUTIONS, THESE KINDS OF
THINKING ABOUT WHAT YOUR
COMMUNITY NEEDS, ARE SO
BEAUTIFUL AND, YOU KNOW, YOU
CAN'T MAKE ONE SIZE THAT'S GOING
TO FIT EVERYTHING EITHER.
THEY HAVE TO BE DONE CLINIC BY
CLINIC BY CLINIC.
WE CAN GIVE A LOT OF GREAT
EXAMPLES.
THANK YOU SO MUCH FOR YOUR
COMMENTS.
>> THANK YOU.
WE HAVE MATTIAS AND PAUL AND
WE'LL PAUSE FOR A MOMENT.
MATTIAS, PLEASE.
>> THANKS, MONICA, FOR MAKING
TIME TODAY.
YES, VERY EXCITING TO SEE YOU.
I CANNOT SHARE MORE, I'M SIGNING
OFF COUNCIL AS WELL, THE LAST
COUPLE YEARS, THIS IS BREAKING
OUR SYSTEM AND WE HAVE
INFLUENCED MANY PEOPLE,
INCLUDING MY OWN CLINIC.
ALSO FASCINATING, MARSHALL
ALREADY INDICATED THAT IN THE
RARE DISEASE SPACE WHERE WE TURN
THINGS UPSIDE DOWN IN NCATS FOR
THE LAST 25 YEARS, WE LET
PATIENTS TRY WHAT WE DO, YOU'LL
TURN THINGS AROUND IMMEDIATELY.
IF PATIENTS CAN TELL US WHAT WE
DO, HOW WE DO IT, AND ARE PART
OF OUR LEADERSHIP TEAM
THROUGHOUT THE PROCESS, THE
ISSUE GOES AWAY AND INVERSES,
OUR PATIENTS ARE TALKING TO THE
STAKEHOLDERS AND MAKE SURE THAT
WHAT WE DO IS UNDERSTOOD AND
TAKEN UP.
AND THE CHALLENGE WILL BE TO
BRING THAT SOCIETY SEGMENTS
WE'RE NOT REACHING WELL BUT AS
STATED, IT'S EXCITING TO SEE IN
ALL OF US WE'RE DOING IT ALREADY
HERE, THIS LITTLE THING, TAKE
THE PATIENTS IN CHARGE, YES, OUR
HEALTHCARE SYSTEMS CMOs MAY
NOT LIKE IT BUT I'LL SEND MY
DATA WHERE I WANT MY DATA SEEN
AND VICE VERSA, SCIENTISTS CAN
REACH PATIENTS WHERE THEY ARE.
SO BUILD THAT IN, AND I'M A
NEPHROLOGIST AT MICHIGAN, THE
KIDNEY POSITION, RARE TO COMMON
DISEASE, OUR PATIENTS DONATING
RISKS TO RESEARCH UNPRECEDENTED.
WE HAVE GOOD PEOPLE IN THIS
COUNTRY IF WE MEET THEM WHERE
THEY ARE AND BUILD A COMMUNITY
CONSENSUS THAT WE CAN MAKE A
DIFFERENCE.
SO YES, FOCUS ON THE TRUST
ISSUE, LET'S EMPOWER OUR
PATIENTS IN SCIENCE TOGETHER AND
HOPEFULLY GET ACROSS A HURDLE
WHICH IS A HUGE CHALLENGE AND
ONLY OUR PATIENTS CAN SOLVE THAT
WITH US.
>> WELL, I'M SURE BASED ON THIS
APPROACH YOUR PATIENTS ARE VERY
FORTUNATE, THEIR OUTCOMES ARE
VERY MUCH MORE SUCCESSFUL
BECAUSE OF THE WAY YOU HAVE
APPROACHED THEM.
SO CONGRATULATIONS.
THAT REALLY IS WHAT PAYS OFF FOR
THEM.
>> THANK YOU VERY MUCH FOR YOUR
COMMENTS, PAUL?
>> YES, THANK YOU SO MUCH FOR
SHARING YOUR VISION.
IT'S VERY EXCITING.
ECHO THE PRAISE FROM EVERYONE
ELSE.
I LOVED THE FOCUS ON TRUST.
THAT'S SUCH A PROBLEM THAT WE
HAVE IN OUR SOCIETY RIGHT NOW.
REALLY APPRECIATE THAT YOU'RE
FOCUSING THERE.
ONE OF THE AREAS THAT YOU
MENTIONED, YOU KNOW, FOCUSED ON
RETURN OF VALUE TO THE
POPULATION RESEARCH
PARTICIPANTS, THAT'S A HUGE
DEAL.
PARTICULARLY IF WE START
THINKING ABOUT THE FACT THAT
DIVERSE COMMUNITIES HAVE DIVERSE
OPINIONS ON WHAT'S VALUABLE TO
THEM.
SO I THINK THAT'S A GREAT AREA
TO FOCUS ON.
AND ANOTHER THING THAT I'M
CURIOUS, YOUR THOUGHTS ON, WOULD
BE BUILDING TRUST IN THESE --
TRUST THROUGH MAKING ACCESSIBLE
SOME OF THESE DATA SOURCES THAT
YOU'RE MENTIONING, AND THAT YOU
WANT TO BUILD.
MAKING THAT DATA AVAILABLE IN A
VERY SAFE WAY, TO EDUCATORS, AND
STUDENTS.
YOU KNOW, AT ALL LEVELS OF
EDUCATION, USING SORT OF REAL
WORLD INFORMATION AROUND
REAL-WORLD PROBLEMS, HEALTH
PROBLEMS THAT PEOPLE HAVE TO
SORT OF TEACH AT THAT LEVEL AND
GAIN AN APPRECIATION FOR DATA
AND DATA METHODS.
CAN YOU SAY A LITTLE BIT MORE
ABOUT YOUR THOUGHTS ON USING
THIS FOR EDUCATION PURPOSES?
>> OH, CERTAINLY.
WELL, SO NATIONAL LIBRARY OF
MEDICINE, YOU KNOW, WE HAVE THIS
ENTITY THAT WE CAN USE AS A REAL
HOME FOR MANY OF THESE
INITIATIVES.
ONE OF ITS MAIN FOCUSES IS GOING
TO BE EDUCATION, ACCESS, BEING
ABLE TO PUT THE TOOLS IN THE
HANDS OF PEOPLE WHO CAN ACTUALLY
USE THEM.
LET ME GO THE OTHER MILE AND
TALK ABOUT THE EDUCATION AND THE
DOCTOR/PATIENT RELATIONSHIP AND
HOW THAT CAN BE INCREDIBLY
HELPFUL SINCE WE'RE TALKING
ABOUT TRUST.
WOULDN'T IT BE AMAZING AND THIS
IS PART OF WHAT WE'RE TRYING TO
BUILD.
WOULDN'T IT BE AMAZING IF YOU
HAD YOUR PATIENTS COME IN AND
SAY, HERE'S MY DATA, AND THEN
YOU, AT YOUR COMPUTER, THE NEXT
TIME YOU SIT DOWN WITH YOUR
PATIENT, CAN SAY, HERE'S THE
DATA COLLECTION WHERE YOUR DATA
IS LIVING, AND HERE IS THE
RESULTS THAT WE'RE SEEING OUT OF
IT.
YOU KNOW, YOU CAN DO THAT ONE ON
ONE WITH THEM, AND THIS IS A
LEARNING THAT WE'RE GETTING, YOU
KNOW, AT THE TIME AND
CONVERSATIONS ABOUT WHAT THE
DISEASE MEANS, WHAT'S THE
QUESTION, WHAT ARE WE TRYING TO
CONQUER, YOU KNOW, THIS -- NOT
ONLY -- WE THINK ABOUT THIS NOT
ONLY IN TRYING TO DEVELOP A
RELATIONSHIP WITH THE PATIENTS
BUT ALSO TRYING TO ENABLE AND
PROMOTE THE RELATIONSHIP BETWEEN
THE CLINICIAN AND THE PATIENT
THEMSELVES AS BEING ABLE TO
LEARN TOGETHER FROM THIS.
>> RIGHT.
THANK YOU.
>> IF I MIGHT ADD TOO, PAUL,
WHILE WE'RE ON THIS TOPIC I'M
REALLY GLAD YOU BROUGHT UP THIS
IDEA OF EDUCATION BECAUSE WHEN
WE'RE TALKING ABOUT THE
COMPLEXITY OF THE DATA THAT
WE'RE TALKING ABOUT, IT'S HUGE.
AND I THINK WE'RE STILL LEARNING
HOW TO HANDLE IT, HOW TO COLLECT
IT.
AND FOR -- IN MY PRESENTATION I
TALKED ABOUT THE NEW TENANT
MODELS THAT WE'RE PROPOSING, AND
DOING FEASIBILITY STUDIES,
PILOTING THEM.
ONE OF THOSE IS EXACTLY ON AN
EDUCATION TENANT.
AND IT'S PARTICULARLY TO TRY TO
ENCOURAGE, YOU KNOW, NEW TO DATA
SCIENCE PEOPLE, ALSO PEOPLE WHO
HAVE BEEN IN MEDICAL SCIENCE
THEIR ENTIRE LIVES BUT HAVEN'T
DONE A LOT IN DATA SCIENCE, AND
ALSO THE POWER USERS.
WE WANT TO REALLY CROSS THE
GAMUT THERE.
BUT THAT'S REALLY THE ONLY WAY
WE'RE GOING TO LEARN ABOUT
WHAT'S NEEDED FOR THEN MAKING
THOSE DATA AS USABLE AS POSSIBLE
FOR THE PEOPLE WHO WANT TO USE
THOSE QUESTIONS.
>> THIS IS REALLY GOOD.
WE'VE SEEN A MICROCOSM WITH THE
CCDI, IT'S IN ITS FIFTH YEAR OF
OPERATIONS, CONGRESSIONALLY
MANDATED.
EVERY CHILD WITH CANCER IN THE
UNITED STATES, THE DATA ARE
SHARED, SHARED AND THERE'S A
USER COMMUNITY BUILT AROUND IT.
AND, YOU KNOW, ALL OF THESE DATA
ARE AVAILABLE.
THERE'S LEARNING.
WHAT THEY FOUND IN THE FIRST
FOUR YEARS, FIRST OF ALL IT'S
BEEN PHENOMENALLY SUCCESSFUL.
EVERYBODY IS CONTRIBUTING.
IT'S NOT JUST DATA SHARING
EXERCISE.
IT'S NOW AN ENTIRE RESEARCH
DEVELOPMENT TEAM, ALL WORKING
TOGETHER.
IT'S NOW LINKED TO A MOLECULAR
CHARACTERIZATION PROTOCOL, BENCH
WORK COMING IN INCLUDED IN
CLINICAL WORK BUT ONE OF THE
FIRST THINGS WE REALIZED LAST
YEAR AT THIS TIME IT'S ONLY
BEING ACCESSED BY POWER USERS.
THAT'S NOT GOOD.
CCDI SPAWNED A PROGRAM TO
FACILITATE, DONE WITHIN THIS
COMMUNITY THAT WAS ALREADY USING
THE DATA SO INSTEAD OF JUST, YOU
KNOW, KIND OF ARTIFICIALLY
GIVING PEOPLE ACCESS IT WAS
SAYING WHAT'S YOUR QUESTION AND
ACTUALLY HELPING THEM GET INTO
THE QUESTION AND ANSWER -- YOU
KNOW, START ENGAGING THEIR
RESEARCH SO WE COULD SEE MUCH
MORE OF THIS HAPPENING.
>> RIGHT.
THAT SOUNDS WONDERFUL.
I THINK THE MORE WE CAN GET SOME
OF THE DATA, THE DATA METHODS
INTO DIDACTIC COURSES THE
EARLIER IN THE EDUCATION
PROCESS, THE BETTER.
>> THANK YOU.
>> JONI, THANK YOU SO MUCH FOR
INVITING ME.
THIS HAS BEEN WONDERFUL.
AND LET ME JUST ALSO THANK YOU
AGAIN SO SINCERELY FOR YOUR
SERVICE ON THIS COUNCIL.
THESE ARE ABSOLUTELY CRITICAL TO
WHAT WE DO, AND BELIEVE ME, ALL
OF THESE RESULTS OF THESE
COUNCIL MEETINGS CRAFT WHAT WE
DO EVERYWHERE AT NIH SO WE'RE
GRATEFUL FOR YOUR INPUT.
>> THANK YOU SO MUCH, MONICA.
WE'LL SEE YOU LATER.
>> BYE-BYE.
>> ALL RIGHT.
LET'S SEE.
SOUNDS LIKE A GREAT WAY TO SORT
OF END THE FIRST PART OF OUR
COUNCIL AFTERNOON.
I'D LIKE TO OPEN FOR ANY
QUESTIONS PERHAPS THAT WE DIDN'T
GET TO BEFORE, SO WE CAN
ACCOMMODATE MONICA.
OR IF YOU'D LIKE TO TAKE A BREAK
AND COME BACK WITH QUESTIONS
BEFORE WE GET STARTED ON THE
NEXT PART THAT'S FINE TOO.
THAT'S YOUR -- SHALL WE TAKE A
FEW QUESTIONS IF THERE ARE ANY
NOW AND MAYBE TAKE A BREAK AT
2:30?
OR ARE YOU READY FOR A BREAK?
I DON'T SEE ANY HANDS UP.
>> LET'S HAVE A BREAK.
>> LET'S HAVE A BREAK, ALL
RIGHT.
THANK YOU, SERGIO.
I LIKE THE DECISIVENESS.
LET'S HAVE A BREAK ANDS WE'LL
COME BACK AT, GOSH, REMIND ME,
GREG, WHEN ARE WE COMING BACK?
>> 2:35.
>> WE'LL SEE YOU SOON.
THANK YOU, EVERYONE.
WELCOME BACK.
AND WE HAVE TWO MORE
PRESENTATIONS TODAY, AND I HOPE
A GOOD DISCUSSION AFTER THESE
PRESENTATIONS AS WELL.
THE FIRST UP WILL BE DAN TAGLE,
DIRECTOR OF OFFICE OF SPECIAL
INITIATIVES, TO GIVE AN UPDATE
ON WHAT'S GOING ON IN HIS AREA.
SO, DAN, I'LL TURN IT OVER TO
YOU.
>> THANK YOU, JONI.
GOOD AFTERNOON, EVERYONE.
SPECIALLY TO ADVISORY COUNCIL
MEMBERS.
I'LL BE GIVING AN UPDATE ON
OFFICE OF SPECIAL INITIATIVES
AND THE PROGRAMS AND ACTIVITIES
THAT WE'RE DOING WITHIN THE
OFFICE.
JUST TO GIVE YOU SORT OF A BRIEF
OVERVIEW, WHO WE ARE, WE'RE A
VERY DIVERSE AND
MULTI-DISCIPLINARY TEAM OF
SCIENTISTS , ALL SEEKING TO
ADDRESS TRANSLATIONAL PROBLEMS
WITHIN NCATS THROUGH INNOVATIVE
SOLUTIONS BY DEVELOPING AND
IMPLEMENTATION DISRUPTIVE
TECHNOLOGIES TO RESULT IN A
PARADIGM SHIFT IN THE FIELD.
TO GIVE YOU A SENSE OF WHAT
WE'RE DOING, ACTIVITIES SPAN
RANGE OF FUNDAMENTAL RESEARCH TO
DEVELOPMENT, CLINICAL
DEVELOPMENT, LEADING TO
REGULATORY ACCEPTANCE.
ALL THE PROGRAMS WITHIN THE
OFFICE ADHERES TO THE NCATS
PRINCIPLES OF DEVELOPING
TECHNOLOGY, DEMONSTRATING
UTILITY, AND THEN ULTIMATELY
DISSEMINATING THE TECHNOLOGY FOR
USE BY THE COMMUNITY.
SO WE HAVE A NUMBER OF DIFFERENT
PROGRAMS, VARIOUS STAGES, ALONG
THE 3D PRINCIPLES, FROM
DEVELOPMENT THROUGH
DISSEMINATION.
COLOR CODED IN THE SENSE LIGHT
BROWN WOULD BE A.I. DRIVEN
MANUFACTURING TECHNOLOGIES BY
START AND ASPIRE.
SCENT PROGRAM AND RADx RAD,
DIAGNOSTIC TECHNOLOGIES.
WE HAVE A NEW PROGRAM, JUST IN
THE EARLY STAGES OF BEING
DEVELOPED, QUANTUM INFORMATION
SCIENCES, SHOWN IN PINK.
AND THEN IN YELLOW A PROGRAM ON
EXTREME, EXOSOME THERAPY FOR
REGENERATIVE MEDICINE, TWO IN
BLUE ARE COLLABORATIVE EFFORTS,
LIGHT BLUE IS COMMON FUND
PROGRAMS, COMMON-FUND-SUPPORTED,
DARK BLUE IS NSF COLLABORATIONS
ON CYBERPHYSICAL SYSTEMS,
NATIONAL ROBOTICS.
IN GREEN IN VITRO MODELING
TECHNOLOGIES, 3D BIOPRINTING AND
TISSUE CHIPS, MICROPHYSIOLOGIC
SYSTEMS.
IT WILL BE A FOCUS OF MY
DISCUSSION ON TISSUE CHIPS, DRUG
SCREENING, TO GIVE A SENSE OF
WHERE THINGS ARE AT IN VARIOUS
PROGRAMS, START, THE PROGRAM
LEAD IS DR. REDNIKE, USING
SYNTHETIC BIOLOGY APPROACHES FOR
PRODUCTION OF COMPOUNDS OR
DELIVERY OF THERAPEUTICS.
ASPIRE INVOLVES COLLABORATION
WITH NCATS INTRAMURAL SCIENTISTS
AND EXTRAMURAL SCIENTISTS,
PRIMARILY TO ESTABLISH PHYSICAL
AND VIRTUAL MODULES FOR
AUTOMATED SYNTHETIC CHEMISTRY,
ARTIFICIAL INTELLIGENCE, MACHINE
LEARNING, ENGINEERING,
INFORMATICS, BIOLOGICAL TESTING
TESTING.
A PROGRAM IN DIAGNOSIS, ASCENT,
STANDS FOR SENSING CONDITIONS
WITH TRONIC TECHNOLOGY, USING
VOLATILE ORGANIC COMPOUND
DETECTION, THIS IS LED BY DR.
CROUCHER AND DR. JOHNSTONE, AND
WE HAVE TWO RADx RAD PROGRAMS,
AGAIN ONE USING ELECTRONIC NOSE
TECHNOLOGY FROM SKIN AND BREATH
TO DETECT SIGNATURES FOR
SARS-COV-2 AND LONG COVID, AGAIN
LED BY DR. CROUCHER AND DR.
JOHNSTONE, AND THEN ONE ON
EXOSOME-BASED ISOLATION
TECHNOLOGIES, PRIMARILY TO
DETECT SARS-COV-2 AND HOST
IMMUNE RESPONSE, AND THIS IS LED
BY DR. HAPPEL AND DR. JOHNSTONE.
WE HAVE THE QUANTUM INFORMATION
SCIENCES, WHICH IS TAKING
ADVANTAGE OF LATEST DEVELOPMENTS
IN QUANTUM TECHNOLOGIES,
PRIMARILY TOWARDS EARLY
DETECTION DIAGNOSTICS AND DEVICE
DEVELOPMENT AND DRUG DEVELOPMENT
LED BY DR. SENTEEL.
THEN THE LAST GROUP WOULD BE ON
EXOSOME-BASED TECHNOLOGY,
INDUSTRIALIZATION AND
TRANSLATION OF EXTRACELLULAR
VESICLES FOR USE IN REGENERATIVE
MEDICINE LED BY DR. HAPPEL, AND
3D BIOPRINTING PROGRAM IS
ANOTHER ONE OF THOSE THAT
INVOLVES NCATS INTRAMURAL
INVESTIGATORS AND EXTRAMURAL
SCIENTISTS, PRIMARILY TO DEVELOP
AND DEMONSTRATE USE OF 3D
BIOPRINTED MATERIALS FOR DISEASE
MODELING AND DRUG SCREENING,
THIS PROGRAM IS LED BY DR.
RUDNICKI.
I'LL BE FOCUSING ON THE ONE
CLOSEST IN TERMS OF MATURITY AS
A PROGRAM, SO THE BIG PROBLEM
THAT WE'RE TRYING TO ADDRESS
HERE WITH TISSUE CHIPS,
MICROPHYSIOLOGICAL SYSTEMS,
DEVELOPMENT OF BETTER PREDICTIVE
TOOLS AND DRUG DEVELOPMENT.
PERCENTAGE OF DRUGS ENTERING
CLINICAL TRIALS IS QUITE ABYSMAL
IN THE SENSE ONLY 10% ARE
SUCCESSFUL, 55% ATTRITION DUE TO
LACK OF EFFICACY, 28% DUE TO
TOXIC EFFECTS IN HUMANS.
ON THE WHOLE IT TAKES AN AVERAGE
TIME OF 10-15 YEARS TO BRING A
DRUG TO MARKET AT A COST OF
$2.6 BILLION.
AND AS SHOWN IN THE CHART WOULD
BE THE AVERAGE IN BLUE, THE TOP
CHART, 9.6%, ACROSS ALL
INDICATIONS WHICH IS THE AVERAGE
OF 10% SUCCESS RATE, WE DO WELL
IN SOME INDICATIONS AND NOT TOO
WELL IN MOST OF THEM.
AND IT'S LARGELY CONSIDERED THAT
THE CURRENT MODELS THAT WE USE
SUCH AS 2D CELL CULTURE SYSTEMS
AND IN SOME CASES IN VIVO ANIMAL
MODELS DO NOT ALWAYS PREDICT
HUMAN RESPONSE.
THAT'S BECAUSE AS SHOWN HERE
THERE'S A LACK OF CONCORDANCE
WITHIN TOXICITIES THAT TENDS TO
BE VERY TISSUE SPECIFIC.
TO GIVE A SENSE OF WHERE THINGS
ARE AT, IN THE TISSUE CHIPS
PROGRAM, THIS IS THE PROGRAM
THAT STARTED BACK IN 2012, JUST
WHEN NCATS WAS BEING STARTED.
AND SO CERTAINLY OVER THE COURSE
OF SEVERAL COUNCILS HAVE GIVEN
UPDATES HOW WE'RE DEVELOPING
TECHNOLOGY, SO I WON'T BELABOR
AND GO OVER THE PROGRESS WE'VE
MADE IN THOSE AREAS.
INSTEAD THIS UPDATE WILL FOCUS
ON LATTER HALF, LATTER
TWO-THIRDS OF THE 3D IN TERMS OF
DEMONSTRATING UTILITY AND
DISSEMINATING THE TECHNOLOGIES
FOCUSED MORE ON HOW WE'RE
DEPLOYING TECHNOLOGY FOR USE IN
PERSONALIZED MEDICINE, HOW WE'RE
BUILDING CONFIDENCE IN THE
TECHNOLOGY, AS SHOWN HERE, AND
HOW WE'RE WORKING TOWARDS
REGULATORY ACCEPTANCE AS WELL AS
BUILDING COMMUNITY AND THEN JUST
AS INDICATION OF THE
PARTNERSHIPS THAT WE HAD, NCATS
IS ALL ABOUT PARTNERSHIPS AS
WELL SO WE HAVE 18 INSTITUTES
AND CENTERS AT THE NIH WHO ARE
PARTNERS IN THIS AS WELL AS
OTHER FEDERAL AGENCIES SUCH AS
FDA, NASA, DARPA, BARDA, EPA, AS
WELL AS PRIVATE PARTNERSHIPS
WITH IQ MPS AFFILIATE, WHICH IS
AN ORGANIZATION MADE OF 26
PHARMA COMPANIES.
NOW IN TERMS OF DEMONSTRATING
UTILITY ARE TISSUE CHIPS A
BETTER PREDICTOR OF HUMAN
PHYSIOLOGIC RESPONSE, SO IN THIS
ILLUSTRATION I'LL FOCUS ON THE
LIVER.
YOU MAY KNOW THE LIVER IS
RESPONSIBLE FOR CONCENTRATING
METABOLIZING MAJORITY OF
MEDICATIONS, AND BECAUSE OF THAT
DRUG INDUCED LIVER INJURY IS THE
MOST COMMON CAUSE OF ACUTE LIVER
FAILURE, 15 TO 20 PER 100
100,000, ADVERSE DRUG REACTION
RESULTING IN LIVER INJURY
REQUIRES DISCONTINUATION OF THE
DRUG, HOSPITALIZATION, LIVER
TRANSPLANT OR EVEN DEATH.
SO HERE'S AN EXAMPLE OF WHAT
LIVER ON A CHIP LOOKS LIKE.
HUMAN HEPATOCYTES AND
NON-PARENCHYMAL CELLS, SO IN
THIS EXAMPLE WE'RE LOOKING AT
PARTICULAR DRUG, WHEN DEVELOPED
ANTI-VIRAL AGENT AGAINST
HEPATITIS B, RAN THROUGH RAT AND
DOG IN VIVO MODELS, AND SHOWED
CLEAR SAFETY, SO WAS APPROVED BY
THE FDA TOWARDS HUMAN CLINICAL
TRIAL USE, BUT UNFORTUNATELY
FIVE OUT OF THE 15 PATIENTS
ENROLLED IN PHASE 1 STUDIES
DIED, DUE TO FORMATION OF --
ACCUMULATION OF FATTY DEPOSITS
IN THE LIVER.
THE QUESTION WE'RE ASKING NOW
COULD HUMAN LIVER ON A CHIP HAVE
PREDICTED TOXICITY RAT AND DOG
MODEL DID NOT.
HERE IS ON THE CHART ON A SHOWS
A RAT HEPATOCYTE, RAT LIVER ON A
CHIP, TESTED ALONGSIDE HUMAN
LIVER ON A CHIP, AND THOSE WITH
ZERO, 1, 10, 30 MICROMOLARS OF
FIALURIDINE, STAINED WITH NILE
RED TO SHOW ACCUMULATION OF
FATTY DEPOSITS SHOWN IN YELLOW.
AS YOU CAN SEE IN THE
QUANTITATION ON THE RIGHT-HAND
SIDE, THE RAT DELIVER HEPATOCYTE
DID NOT SHOW ANY SIGNIFICANT
STAINING WITH NILE RED, WHEREAS
IN HUMAN HEPATOCYTE CHIP,
INDICATES INCREASE IN FATTY
DEPOSIT WHICH TRANSLATES.
ONE CAN LOOK AT FUNCTIONAL
MARKERS SUCH AS ALBUMIN
SECRETION, SHOWING AT HIGH DOSE
OF FIALURIDINE ALBUMIN
SECRETION, HUMAN HEPATOCYTE
SHOWED DECREASE DEPENDENT,
LOOKING AT LIVER INJURY MARKERS,
THERE'S ALSO A DOSE-DEPENDENT
INCREASE IN EXPRESSION OF THOSE
LIVER INJURY MARKERS.
THIS STUDY WAS ALSO EXPANDED TO
A GROUP OF 22 OTHER COMPOUNDS,
JUST LIKE FIALURIDINE, THAT WERE
TESTED IN ANIMAL MODELS, 2D CELL
CULTURE MODELS, AND PASSED
THROUGH THE INITIAL PRE-IND
SUBMISSION AND THEN WENT INTO
CLINICAL TRIALS, BUT
UNFORTUNATELY OUT OF THE 22
COMPOUNDS THAT WERE TESTED JUST
LIKE FIALURIDINE THEY ALL FAILED
IN HUMAN TRIALS AND SUBSEQUENTLY
WITHDRAWN DUE TO TOXICITIES
RESULTING IN MORE THAN 200
PATIENT DEATHS, TEN LIVER
TRANSPLANTS, WHEN TESTED.
WHEN HUMAN LIVER ON A CHIP WAS
LOOKED AT, WITH THIS COMPOUND,
THE HUMAN LIVER ON A CHIP SHOWED
87% SENSITIVITY, AS WELL AS 100%
SPECIFICITY, BEING ABLE TO
PREDICT THE DRUG TOXICITIES THAT
THE OTHER MODELS WERE NOT ABLE
TO PREDICT.
NOW SHIFTING GEARS A LITTLE BIT
IN TERMS OF HOW WE CAN
DEMONSTRATE USE OF TISSUE CHIPS
FOR DISEASE MODELING AND
EFFICACY STUDIES, HERE IS AN
EXAMPLE A GROUP OF RARE DISEASES
WHICH ARE AUTOIMMUNE
DEMYELINATING NEUROPATHIES
SUCHY POLY NEUROPATHY AND MOTOR
NEUROPATHY HAVE NO PARTICULAR
MOD TOLLS RELY ON, BUT IT'S AN
AUTOIMMUNE DISEASE, SO THE
PATIENTS DO DEVELOP ANTI-SERA,
AGAINST THEIR OWN NEUROMUSCULAR
JUNCTION, SO THE TISSUE CHIP
MODEL HERE CONSISTS OF A
CO-CULTURE OF HUMAN PRIMARY
SCHWANN CELLS, AND INDUCED
PLURIPOTENT STEM CELL-DERIVED
MOTONEURONS.
WHEN TREATED WITH ANTI-SERA THAT
WAS ISOLATED FROM 15 PATIENTS,
EACH OF THESE BOXES ON THE
HISTOGRAM RECOMMENDS ANTI-SERA
FROM EACH, EACH JUNCTION ON A
CHIP WHEN STAINED WITH, AGAIN,
SHOWED YOU CAN GRASP EACH OF THE
ANTI-SERA ON JUNCTION SHOWED
STAINING FOR THIS COMPLEMENT
ACTIVATION PATHWAY.
NOW, TO TEST FOR EFFICACY OF
CANDIDATE DRUG TNT005, A
MONOCLONAL ANTIBODY, WHEN
TREATED SHOWED THAT THEY DID NOT
GET ACTIVATED OR ACCUMULATE IN
THE PATIENT'S SERA.
EFFICACY DATA WAS INCORPORATED
WITH OTHER DATASETS, AND WAS
THEN SUBMITTED TO THE FDA IND,
NOW IN PHASE 2 SPONSORED BY
SANOFI.
ANOTHER AREA DEMONSTRATING
UTILITY OF TISSUE CHIPS IS
MODELING DISEASES THAT ARE HARD
OR DIFFICULT TO MODEL HERE ON
EARTH, ESPECIALLY TAKES LONG
TIME SO ONE AREA OF COURSE IS IN
AGING.
AS IT TURNS OUT, UNDER THE
INFLUENCE OF A MICROGRAVITY
ENVIRONMENT ONE CAN ACCELERATE
EFFECTS OF AGING AT LEAST
OBSERVE THE CLINICALLY WITH
ASTRONAUTS, THE QUESTION IS CAN
WE CAPTURE THOSE PHYSIOLOGICAL
CHANGES THE ASTRONAUTS' CELLS
AND TISSUES EXPERIENCE UNDER
MICROGRAVITY AND BE ABLE TO TAKE
ADVANTAGE OF THE MICROGRAVITY
ENVIRONMENT AND BE ABLE TO
DEVELOP MODELS OF ACCELERATED
AGING AND BE ABLE TO ACHIEVE
THAT, SO WE HAVE PROJECTS THAT
LOOK AT IMMUNOSENESCENCE,
CHANGES IN BLOOD-BRAIN BARRIER
PERMEABILITY, RESULT OF EXPOSURE
TO MICROGRAVITY, THE
MOBILIZATION OF IMMUNE SYSTEM
FROM BONE MARROW AS A
CONSEQUENCE OF LUNG INFECTION,
LOOKING AT OSTEOARTHRITIS,
PROTEINURIA AND KIDNEY STONE
FORMATION, ALSO CARDIAC
DYSFUNCTION, ALSO LOOKING AT
MUSCLE WASTING OR SARCOPENIA,
AND CHANGES IN THE GUT
MICROBIOME, RESULTING IN GUT
INFLAMMATION.
SO HERE'S AN EXAMPLE OF AN
ENDORSEMENT I GUESS FROM THE
ASTRONAUTS WHO WORKED WITH
TISSUE CHIPS.
>> GREETINGS FROM THE
INTERNATIONAL SPACE SPACE
STATION, I'M HERE WITH MY CREW
MATE OF THE JAPAN AGENCY, MY
CREWMATES AND I HAVE BEEN HARD
AT WORK SUPPORTING AN ARRAY OF
RESEARCH EXPERIMENTS AND
TECHNOLOGY DEMONSTRATIONS IN
THIS MICROGRAVITY ENVIRONMENT,
SPACE STATION IS UNIQUE
LABORATORY UNLIKE ANY OTHER
WHERE THERE'S NO GRAVITY.
AND BECAUSE OF THAT WE CAN LOOK
AT SCIENCE IN A NEW WAY TO
BENEFIT HUMANITY.
>> ONE OF THE AREAS OF INTEREST
IS RESEARCH IN HUMAN CELLS AND
TISSUES, THROUGH A LAB ON A
CHIP, WHAT WE LIKE TO CALL
TISSUE CHIPS.
WE KNOW THAT THE SPACE
ENVIRONMENT HAS PROFOUND IMPACT
ON HUMAN PHYSIOLOGY, AND THROUGH
TISSUE CHIPS WE CAN SEEK NEW
AVENUES FOR DRUG DISCOVERY,
POTENTIAL THERAPEUTIC
DEVELOPMENT IN THE SPACE
ENVIRONMENT.
MANY OF THESE TISSUE CHIP
INVESTIGATIONS ARE NEAR AND DEAR
TO THE AMERICAN HEART
ASSOCIATION.
>> I ENCOURAGE THOSE IN
ATTENDANCE TO THINK ABOUT TAKING
YOUR RESEARCH BEYOND EARTH'S
HORIZON, THROUGH SPACE-BASED
RESEARCH WE CAN HOPEFULLY FIND
WAYS TO TREAT MANY MITIGATING
AILMENTS HUMANS DEVELOP ON OUR
PLANET.
THANK YOU FOR ALL YOU DO TO
TREAT PATIENTS ON EARTH E EARTH.
>> THAT'S RAISING IT TO A WHOLE
NEW BAR.
IF YOU'RE GOING TO START DOING
MESSAGES FROM SPACE NOW, COME
ON!
>> WELL, WHAT CAN WE SAY?
WE'RE INNOVATIVE, OUTSIDE OF THE
BOX, OUTSIDE OF EARTH THINKERS,
YES.
JUST TO GIVE A SENSE OF HOW THE
EXPERIMENTS IN LOW EARTH ORBIT,
LEO, IS BENEFITING RESEARCH, SO
WE HAVE HAD CERTAINLY ABOUT NINE
PAYLOADS SENT OUT INTO
INTERNATIONAL SPACE STATION, AND
SO FAR, YOU KNOW, SO FAR WE'VE
BEEN ABLE TO CAPTURE
AGING-RELATED MARKERS.
FOR EXAMPLE, CARDIOVASCULAR
DECONDITIONING, SIX MONTHS OF
EXPOSURE UNDER MICROGRAVITY IS
EQUIVALENT TO ROUGHLY TEN YEARS
OF BIOLOGICAL AGING, BUT WHEN WE
SENT TISSUE CHIPS INTO THE SPACE
STATION WITHIN SIX WEEKS,
CERTAINLY FOUR WEEKS TO SIX
WEEKS, THERE IS ALREADY
DECREASED CONTRACTILITY, TWITCH
FORCE, SIMILAR TO THOSE IN AGING
ON MUSCLE CELLS, BUT ALSO IMMUNE
CELLS, BONE AND SKELETAL
MUSCLES.
ONE OF THE MAIN BENEFITS WE'VE
LEARNED WORKING WITH NASA
ENGINEERS AND PAYLOAD DEVELOPERS
WE CAN TAKE ADVANTAGE OF
DEVELOPMENT IN PARTNERSHIP WITH
THOSE PAYLOAD DEVELOPERS IN
IMPROVING TECHNOLOGIES FOR MPS,
BECAUSE IT TAKES A VERY SMALL
FOOTPRINT TO SEND CARGO TO
SPACE, AND SO BECAUSE OF THAT WE
HAVE TO MINIATURIZE AND AUTOMATE
OUR PLATFORM SENDING THEM TO
SPACE.
THE BENEFIT OF MINIATURIZATION
AND AUTOMATION CAN CERTAINLY
BENEFIT USE OF TISSUE CHIP
TECHNOLOGY ON EARTH, SO WE HAVE
RELEASED TWO RFAs CURRENT
RIGHT NOW SO THAT WE CAN MAKE
TISSUE CHIP TECHNOLOGY TO BE
COST EFFECTIVE AS WELL AS
AVAILABLE TO MANY RESEARCHERS
HERE ON EARTH.
CERTAINLY IN MY PRESENTATION FOR
CONCEPT CLEARANCE AT COUNCIL
BACK IN SEPTEMBER I MENTIONED
THAT MODERN DISEASES, HUMAN
CONDITIONS ON EARTH -- MODELING
CAN BE DIFFICULT, LEO, LOW EARTH
ORBIT, MICROGRAVITY, DEVELOPING
COUNTERMEASURES FOR USE ON EARTH
AND LOOK AT BIOMANUFACTURING
CAPABILITIES SUCH AS PROTEIN
CRYSTALLIZATION, PROTEIN CRYSTAL
TENDS TO FORM PERFECT CRYSTALS
UNDER MICROGRAVITY, WE CAN LOOK
AT 3D BIOMETRIC BIOPRINTING TO
BIOPRINT PERHAPS ENTIRE ORGAN
SYSTEMS THAT CAN BE USED FOR
TRANSPLANTATION AND REGENERATIVE
MEDICINE BEING ABLE TO DEVELOP
THE TELEMETRY OR ROBOTICS,
MEDICAL CAPABILITIES, BECAUSE
MOST OF THE READOUTS WE DO WHEN
WE SEND TISSUE CHIPS TO SPACE
ARE SENT BACK TO US THROUGH
TELEMETRY, ONE AREA OF INTEREST
WOULD BE IN THE DEVELOPMENT OF
LONG SHELF LIFE PHARMACEUTICALS,
EVEN PHARMACOLOGY AND DEMAND
CAPABILITIES.
SO IN THAT REGARD, AS PRESENTED
AS A CONCEPT CLEARANCE LAST
SEPTEMBER WE HAVE RENEWED TISSUE
CHIPS IN SPACE PROGRAM, AND
WE'RE IN THE PROCESS OF WRITING
NOTICE OF FUNDING OPPORTUNITY
ANNOUNCEMENTS FOR THAT.
ONE AREA DEMONSTRATING
USEFULNESS IS PRECISION
MEDICINE, PERSONALIZED MEDICINE.
EXAMPLE IN TERMS OF PROSTATE
CANCER METASTASIS TO BONE
MARROW, METASTATIC RESISTANT
RESISTANT PROSTATE CANCER IS
LETHAL, NO CURES, WE'RE RUNNING
PHASE 2 CLINICAL ARM TRIAL, IMMU
32, ANTIBODY DRUG CONJUGATE
WHICH MARKS MOST RAPIDLY
PROLIFERATING CELLS, AND THIS
ADC IS CONJUGATED TO DRUG SN-38
TOPOISOMERASE INHIBITOR, WHICH
CAUSES APOPTOSIS, RAPIDLY
PROLIFERATING CELLS.
SO THIS IS A STUDY SPONSORED BY
GILEAD.
WE TAKE TUMOR BIOPSIES FROM 50
PATIENTS ENROLLED IN PHASE 1
STUDY, AND PUT IN TISSUE CHIP,
RECREATE TUMOR MICROENVIRONMENT
FOR THE PATIENTS SUMP AS
INCORPORATION OF M2 MACROPHAGES,
OSTEOBLASTS TO IDENTIFY WHICH OF
THE PATIENT POPULATION WILL BE
RESPONDERS AND NON-RESPONDERS
AND THAT'S BEING ABLE TO
EFFICIENTLY STRATIFY THE PATIENT
GROUP FOR MORE EFFICIENT
CLINICAL TRIALS.
ONE AREA THAT WE'RE
DEMONSTRATING UTILITY IN
BUILDING CONFIDENCE IN THIS
TECHNOLOGY SO WHAT WE'VE DONE IS
CREATE VALIDATION FRAMEWORK,
VALIDATION WOULD BE BY
DEVELOPERS OF THE TECHNOLOGY
THROUGH PUBLICATIONS, AND THEN
NCATS FUNDED ESTABLISHMENT OF
TISSUE CHIP TESTING CENTERS FOR
MIT, TEXAS A & M, A FEDERATED
MPS DATABASE THROUGH UNIVERSITY
OF PITTSBURGH, PRIMARILY TO
INDEPENDENTLY TEST RESULTS OF
THE PHYSIOLOGICAL VALIDATION BY
DEVELOPERS IN TERMS OF
DEMONSTRATING ROBUSTNESS,
REPRODUCIBILITY, RELIABILITY AND
RELEVANCE OF THE DATA THAT WAS
GENERATED DURING THE
PHYSIOLOGICAL VALIDATION.
AND NOW WE'RE IN THE LAST STAGE
OF THE VALIDATION FRAMEWORK,
INDUSTRIAL, WHERE WE HAVE
ENCOURAGED TESTING CENTERS IN
THE DATABASE TO FORM
SELF-SUSTAINING BUSINESS MODELS
TO BE ABLE TO CONTINUE EFFORTS
ON THIS PARTICULAR CASE, M.I.T.
BECAME CRO, NOW JAVELIN BIOTECH,
AND TEXASA BECAME MODEL
PARTICIPATING BY ACADEMIA,
INDUSTRY, MPS IS NOW BIOSYSTICS
PLATFORM.
DEMONSTRATING UTILITY WE'VE SEEN
INCREASED USE OF MPS, TISSUE
CHIPS BY PHARMA.
WE PARTNERED WITH THE MPS
AFFILIATE, MADE UP OF 26 PHARMA
COMPANIES, AND THROUGH OUR
PARTNERSHIPS THEY HAVE
CO-AUTHORED A NUMBER OF
PUBLICATIONS INDICATING WHAT ARE
FIT FOR PURPOSE FOR INDUSTRY
USE, IN TERMS OF VARIOUS ORGAN
SYSTEMS, WHAT TYPES OF
BIOMARKERS AND ASSAYS ARE
NEEDED, PARTICULARLY IN THE
AREAS OF HIGH NEEDS WITHIN
PHARMA AND OF COURSE IN DRUG
DEVELOPMENT AS WELL.
HERE IS A SNAPSHOT OF THINGS,
ACTIVITIES DONE BY PHARMA.
HERE ARE THE END USERS,
PRIMARILY THE LARGE
PHARMACEUTICAL COMPANIES, AND
THEN COMMERCIAL COMPANIES THAT
SUPPLY TISSUE CHIPS, AND
DIFFERENT TISSUE CHIPS SHOWN IN
THE COLUMN BY DIFFERENT -- USED
BY DIFFERENT PHARMA COMPANIES,
AND WHAT THEY ARE USED FOR IN
DRUG DEVELOPMENT.
SO PRIMARILY CENTERED WITHIN
TARGET IDENTIFICATION,
PRE-CLINICAL SAFETY,
PRE-CLINICAL EVE CASE, FARM COVE
KINNETT-- PHARMACOKINETICS.
THIS IS IN ADDITION TO BUILDING
CONFIDENCE BY ALLOWING ACCESS TO
THE DATA.
THIS IS ALSO ONE WAY THAT WE'RE
DISSEMINATING THE INFORMATION
WHERE WE CERTAINLY TAKE
EXPERIMENTAL DATA FROM TISSUE
CHIP OR MICROPHYSIOLOGICAL
SYSTEMS, INCORPORATE WITH META
DATA COMING FROM CLINICAL
DATASETS, PRE-CLINICAL DATA, AS
WELL AS COMPOUNDS THAT HAVE
ALL -- UNIVERSAL COMPOUNDS HAVE
BEEN TESTED IN HUMANS.
MOREOVER ON TOP OF THAT,
ANALYTICAL TOOLS HAVE BEEN
DEVELOPED BY THE DATABASE TO BE
ABLE TO BE USED IN TERMS OF
DETERMINING EXPERIMENTAL MODEL
AND REPRODUCIBILITY, COMPOUND
SAFETY AND EFFICACY, AS WELL AS
MECHANISM OF DISEASE, MECHANISM
OF COMPOUND ACTION, BEING ABLE
TO DEVELOP COMPUTATIONAL MODELS
FOR ADME AND TOXICITY AND
DISEASE PATHOLOGY.
ONE WAY WE'RE DISSEMINATING
TECHNOLOGY BY PARTNERSHIP WITH
OFFICE OF STRATEGIC ALLIANCE
ENCOURAGING FORMATION OF
START-UP AND SPINOFF COMPANIES,
IN THIS AREA OF 3D TISSUE
MODELS, AND LARGELY HAVE BEEN
SUPPORTED THROUGH SMALL BUSINESS
FUNDS, NOT ONLY BY NCATS BUT
CERTAINLY BY THE NIH AS WELL.
LASTLY, ONE AREA OF
DISSEMINATION WE'RE TRYING TO DO
IS IN GOING TOWARDS REGULATORY
ACCEPTANCE, SO IN THIS
PARTICULAR ACTIVITY WE HAVE
ESTABLISHED TRANSLATIONAL
CENTERS FOR MICROPHYSIOLOGICAL
SYSTEMS, THIS IS A LETTER OF
AGREEMENT WITH NCATS AND FDA
WITH NOTION THAT WE WOULD FUND
ABOUT THREE TO FOUR CENTERS, ALL
OVER THE UNITED STATES, AND EACH
CENTER WOULD TAKE FOUR TO FIVE
VALIDATED MPS WITH SPECIFIC
CONTEXT OF USE, AND WORK
ALONGSIDE THE FDA IN BEING ABLE
TO DEVELOP QUALIFICATION
SUBMISSION PACKAGE THAT FDA
WOULD REQUIRE TO BE ABLE TO
QUALIFY THESE TISSUE CHIPS AS
DRUG DEVELOPMENT TOOLS.
WHAT WE HAVE ULTIMATELY WOULD BE
TISSUE CHIPS FIT FOR PURPOSE,
FOR INDUSTRY NEEDS, WITH
SPECIFIC CONTEXT OF USE, THAT
MEETS REGULATORY QUALIFICATION,
AND THEN BE ABLE TO RAPIDLY
COMMERCIALIZE THAT SO IT CAN BE
USED BY THE GENERAL RESEARCH
COMMUNITY.
THE LAST YEAR IN TERMS OF
DISSEMINATION IS THROUGH
ESTABLISHMENT OF INTERNATIONAL
USERS THROUGH MPS WORLD SUMMIT,
PRIMARILY WORKING WITH ALL THE
MAJOR STAKEHOLDERS, PHARMA,
REGULATORY AGENCIES, ACADEMICS,
INDUSTRY, TO BE ABLE TO CONNECT,
EXCHANGE, EDUCATE EACH OTHER IN
THE USE OF TISSUE CHIPS, SO
NCATS FUNDED THROUGH SEED
FUNDING CONFERENCE IN NEW
ORLEANS, 2022, AND JUST LAST
YEAR IN BERLIN, GERMANY.
JUST TO SHOW THE INTEREST, BY
THE COMMUNITY, NEW ORLEANS WE
HAD 450 IN-PERSON ATTENDEES,
ALMOST 1300 IN BERLIN, A NUMBER
OF ABSTRACTS THAT HAVE MORE THAN
DOUBLED IN BERLIN COMPARED TO
NEW ORLEANS, SPEAKERS REMAIN
CONSTANT, IT'S A FOUR-DAY
SYMPOSIUM, WE HAVE TO KEEP THE
NUMBER OF SPEAKERS AT THE
OPTIMAL LEVEL, BUT THE NUMBER OF
POSTERS AS WELL WELL AS EARLY
STAGE INVESTIGATORS HAVE GROWN
IN NUMBER AS WELL AS
PARTICIPATING COUNTRIES AND
NUMBER OF SPONSORS.
THE LAST AREA WE'RE DOING TO
TRAIN THE NEXT GENERATION IN
SCIENTISTS, ESTABLISH
INTERNATIONAL MPS SOCIETY WITH A
VISION TO ACCELERATE MPS,
IMPROVE HEALTH AND ENVIRONMENT,
TRAIN THE NEXT GENERATION OF
SCIENTISTS IN THE NEW
TECHNOLOGY, JUST TO BRING TO
YOUR ATTENTION THAT THIS YEAR
WE'RE HAVING OUR THIRD ANNUAL
MEETING IN SEATTLE, JUNE 10-14.
AND THEN BECAUSE OF THE NCATS
INVESTMENT AND ACTIVITIES IN
LEADING THE EFFORTS IN TERMS OF
IN VITRO MODEL, CERTAINLY
THERE'S BEEN A SURGE IN
INTEREST, NOT ONLY WITHIN NIH
BUT ACROSS FEDERAL AGENCIES, AND
ACROSS THE BIOMEDICAL RESEARCH
COMMUNITY IN USE OF NEW
APPROACHES METHODOLOGIES,
NAMs.
AS JONI MENTIONED IN HER
DIRECTOR'S REPORT, THERE'S A NEW
EFFORT CONCEPT WITHIN NIH COMMON
FUND TO LOOK AT THE VARIOUS NAM
APPROACHES, IN SILICO, SUCH AS
IN SILICO MODELING, COMPLEX IN
VITRO, CHEMICAL SENSING, BE ABLE
TO INTEGRATE THAT TOGETHER, TO
FORM LIKE A COMMON DATA
ECOSYSTEM WHERE ALL THE DATASETS
ARE BEING INTEGRATED AND AS JONI
MENTIONED BEING ABLE TO LOOK AT
THE WHOLE BEING BETTER THAN THE
SUM OF ITS PARTS.
AND THEN I'D LIKE TO THANK THE
PROGRAM STAFF THAT ARE VERY
INVOLVED IN THE TISSUE CHIP
PROGRAM.
AND ACKNOWLEDGE ALL THE OTHER
PARTNERS IN THE DIFFERENT
INSTITUTES AND CENTERS AT THE
NIH, PARTNERSHIP WAS THE FOOD
AND DRUG ADMINISTRATION, IQ MPS
AFFILIATE, NASA, BARDA,
INTERNATIONAL SPACE STATION,
NATIONAL LABORATORY.
WITH THAT I'LL END WITH CALL FOR
QUESTIONS, PRIMARILY CERTAINLY
WOULD APPRECIATE COMMENTS FROM
COUNCIL MEMBERS IN TERMS OF FOUR
AREAS THAT I'VE IDENTIFIED IN
TERMS OF WHAT OTHER AREAS OF
BIOMEDICAL RESEARCH APPLICATIONS
CAN NCATS LEVERAGE, TISSUE CHIP
TECHNOLOGIES, ARE THERE
ADDITIONAL STAKEHOLDERS NCATS
SHOULD ENGAGE FOR DISSEMINATION,
AND THIRDLY BEYOND TRADITIONAL
CENTERS FOR MPS DOES COUNCIL
ENVISION OTHER ACTIVITIES TO GET
TO PROMOTE WIDESPREAD ADOPTION,
AND THEN THE LAST BULLET WOULD
BE HOW CAN NIH BUILD UPON NCATS'
INVESTMENT IN TISSUE CHIPS TO
ENHANCE GOAL OFP NIH COMMON
GUNNED COMPLEMENT-ARIE PROGRAM
AND HOW DO WE COORDINATE WITH
INTERNATIONAL FUNDING AND
REGULATORY AGENCIES AND I'LL
ENTERTAIN QUESTIONS FROM
COUNCIL.
>> I SEE MATTIAS AND MARSHALL TO
GET US STARTED.
AS OTHERS THINGS WE'LL START
WITH MATTIAS.
>> CONGRATULATIONS.
YOU HAVE BUILT A FULCRUM, ONE OF
THE DIAMONDS IN THE NCATS
PORTFOLIO.
IT IS REALLY, REALLY IMPRESSIVE.
SO MANY LEVELS.
TO AN EXTENT I HAVE TO SAY WHEN
YOU BUILD OUT YOUR TRIAD WHERE
MPSs ARE KEY SOURCE OF
INFORMATION, ONE ASPECT WHICH I
WOULD RECOMMEND TO CONSIDER IS
ALSO FOR VALIDATION,
REGISTRATION, CONTEXT OF USE
DEFINITION IS LET'S GO BACK TO
OUR PATIENTS, AND PATIENTS AS WE
DISCUSSED EARLIER IF WE ENGAGE
THEM PROPERLY THEY ARE WILLING
TO DO THAT, IN THE SENSE ONE OF
THE MAIN CHALLENGES RIGHT NOW IS
TO BENCHMARK THE CLINICAL TRIALS
ON THE CHIP AGAINST OUR CLINICAL
TRIALS IN HUMANS.
AND THE CHIP FORMS ARE
OUTPERFORMING OUR KNOWLEDGE
ACQUISITION FROM HUMANS BY
ORDERS OF MAGNITUDE, MAYBE
EXCEPT IN THE NEOPLASTIC FIELD.
WITH THE TECHNOLOGIES WHICH WE
SEE RAPIDLY EXPLODING IN EVEN
OUT OF SMALL AMOUNTS OF TISSUE
TO GENERATE MASSIVE AMOUNTS OF
DATA, WHICH WILL ALLOW YOUR
A.I.-DRIVEN DISEASE MODELING TO
USE PATIENT-DERIVED MATERIALS,
IT WILL BE FOR MY FIELD AND
PRECISION MEDICINE CONCEPTS WE
HAVE NOW 18 DIFFERENT HIGHLY
COMPLEX PROFILING TECHNOLOGIES
RUN AGAINST TWO FINE NEEDLE
BIOPSIES FROM PATIENT VOLUNTEERS
WHO DONATE TISSUE RESEARCH IN
CLINICAL CARE AND CLINICAL
TRIALS.
SO, JONI, WHAT MIGHT BE ONE
UNIQUE SPOT FOR NCATS IS THAT IF
YOU COUPLE OPERATIONAL WITH
EFFORT IN THE RARE DISEASE
NETWORKS THAT YOU HAVE ACCESS TO
HIGHLY DEDICATED PATIENT
POPULATIONS, WHO ARE WILLING TO
DONATE THESE TO RESEARCH, THEY
ARE REALLY DOING THIS, EVEN
DONATING BIOPSIES, IN TISSUE
RISKY TO BIOPSY KIDNEY
SUCCESSFULLY AND SAFELY, YOU CAN
DO THE SAME FOR MANY OTHER
CHRONIC DISEASES WITH ORGANS ARE
ACCESSIBLE AND THEY ARE
INNOVATIVE WAYS IF YOU START
WITH SMALL COHORT OF A FEW
HUNDRED PEOPLE WHERE HAVE YOU
THAT KNOWLEDGE, THEN PROJECT OUT
INTO THE EX VIVO SYSTEMS WHERE
CLINICALLY TISSUE ON A CHIP CAN
BE TUNED SO WE KNOW WHERE IT
WORKS AND DOESN'T.
NO OTHER PLACE TO DO THAT THAN
NCATS BECAUSE YOU HAVE THE
PLATFORM AVAILABLE, RDCRN AND
CTSA FOR RARE AND COMMON
DISEASE, BLOOD FORM, IF WE
ENGAGE PATIENTS EARLY THAT THEY
ARE THE STAKEHOLDERS IN OUR RARE
DISEASE NETWORK, MARSHALL, WE
WILL SUPPORT IT, THEY ARE
DRIVING US TOWARDS THESE TYPE OF
APPROACHES, SO PATIENT ADOPTION
IS SIGNIFICANTLY LESS A
CHALLENGE THAN IN THE PAST.
STAY IN TOUCH, PARTICULARLY
ASTHMA, TECHNOLOGY DEVELOPMENT
WHICH ALLOWS US TO FEED INTO
A.I. DRIVEN, IF YOU WOULD HAVE
ASKED ME THREE OR FOUR YEARS AGO
WOULD HAVE BEEN UNIMAGINABLE,
MONITOR STATES OUT OF
CROSS-SECTIONAL ASSESSMENT, AS
WAS BEAUTIFULLY PUBLISHED BUILD
EX VIVO ORGAN ON A BLOOD MODEL
SYSTEMS TO PREDICT TOXICITY OF
ORGAN CROSSTALK, NOT ABLE TO
CAPTURE EARLY.
RAW MATERIALS ARE IN PLACE,
CLEARLY CLINICAL COLLEAGUES
OUTPERFORMED US WITH DATA AND
INFORMATION CAPTURE, BUT
CLINICAL FRAMEWORK IF NCATS IS
WILLING TO DRIVE THAT FRAMEWORK.
>> THANK YOU FOR YOUR COMMENTS
AND SUGGESTIONS.
CERTAINLY THAT'S IN OUR MIND IN
TERMS OF HOW WE ARE MOVING
FORWARD WITH TISSUE CHIPS,
ESPECIALLY WITH POWERED BY THE
NIH COMMON FUND WHERE WE'RE
ACTUALLY LOOKING TO CREATE
DIGITAL TWINS WITH ELECTRONIC
HEALTH RECORDS OF PATIENTS, AND
THEN BEING ABLE TO PAIR THAT
WITH BIOMEMETIC TWINS OR TISSUE
CHIPS SO WE CAN IN SOME WAYS
CROSS-VALIDATE EACH OTHER BOTH
IN TERMS OF THE DIGITAL OR COME
COMPUTATIONAL PREDICTIONS,
LOOKING AT HOW CLOSE
APPROXIMATION WE GET WITH
COMPUTATIONAL OR IN VITRO
VALIDATION.
>> ONE THING I WOULD ADD TOO,
MATTIAS, I THINK YOU'RE
ABSOLUTELY RIGHT.
THAT'S THE DIRECTION, AT LEAST
ONE OF THE DIRECTIONS WE'RE
INTERESTED IN GOING INTO, AND I
MENTIONED THE I3D WORKSHOP, PART
OF THAT IS ALSO TRYING TO ENSURE
WE HAVE SOME SORT OF FRAMEWORK
TO DO THAT, IN ADDITION YOU TALK
ABOUT THE ACCESSIBLE ORGANS, I
THINK THAT'S SUPER IMPORTANT.
WE'VE BEEN USING iPS CELLS,
GENERATING, YOU KNOW, REDERIVED
SPECIFIC CELL TYPES, SO THAT'S
AN IMPORTANT TECHNOLOGY BUT
GETTING PRIMARY CELLS IS ALSO
IMPORTANT IF WE CAN, AND THE
THIRD APPROACH THAT WE'VE BEEN
LOOKING AT IS USING CRISPR TO
THEN CREATE THE MUTATION WE
MIGHT SEE IN A PARTICULAR
DISEASE AREA.
SO THOSE THREE APPROACHES I
THINK, LOOKING AT THEM IN
PARALLEL, DOING THE DEEP SORT OF
TECHNICAL WORK TO EVALUATE ANY
DIFFERENCES WILL TELL US, YOU
KNOW, WHETHER WE LOSE THE
GRANULARITY OR IF THAT
GRANULARITY IS EVEN IMPORTANT.
SO I THINK, YOU KNOW, BEING ABLE
TO DO THOSE KINDS OF STUDIES IS
SOMETHING WE WANT TO ALSO
ESTABLISH AS WELL, ON THAT
COURSE, TO MORE CLINICAL
ENGAGEMENT.
>> JONI, BUILD IN THE CLOSING,
BUILD IN A COHORT WHERE YOU
ACTUALLY TEST YOUR MODELS, AND
YOU WILL SEE WHERE YOUR MODELS
FAIL AND THEN WITH POWER OF THE
MODEL BUILDS YOU HAVE RIGHT NOW,
A FEW ITERATIONS, YOU WILL MAKE
LEAPS FORWARD WITHOUT BENCH
MARKING AGAINST IN VIVO STATE,
IN CLINICAL TRIALS YOU WILL DO
THE DO THAT.
THE TECHNOLOGY IS THERE.
PATIENT INTEREST IS THERE.
YOU HAVE TO PUSH CLINICIAN
SCIENTISTS TO SWITCH INTO
INVASIVE MODE BUT IT CAN AND IS
DONE SAFELY IN MULTIPLE ORGAN
SYSTEMS, MOST OF THE ORGANS ARE
ACCESSIBLE, MAKES A FUNDAMENTAL
DIFFERENCE FOR MODELING TEAMS.
>> AGREED, YES.
>> A PAPER FROM US FROM A COUPLE
DAYS AGO WE BUILD OUT.
>> THAT'S TERRIFIC.
I WOULD LOVE TO SEE THAT.
AGAIN, YOU MIGHT BE ROTATING OFF
BUT WE'LL BE IN TOUCH ON THESE
IDEAS TOO AS THEY GO FORWARD.
I APPRECIATE THOSE COMMENTS.
MARSHALL?
>> TWO COMMENTS AND OBSERVATION.
THE REST OF THE COUNCIL, WHEN WE
ROTATE OFF, YOU HAVE TO STEP UP
AND START RAISING YOUR HANDS,
OTHERWISE IT'S GOING TO GET
QUIET HERE.
AFTER RARE DISEASE WHICH IS
WHERE MY HEART WILL ALWAYS DWELL
THIS IS MY FAVORITE PROGRAM,
BECAUSE IT'S THE BLUE SKY
SECTION OF NCATS, ONE
OBSERVATION IS YOU REALLY NEED
TO START ENGAGING THE FDA
HEAVILY IN THIS, PARTICULARLY
BIOMEDICAL QUALIFICATION
PROGRAM.
BECAUSE WHAT I THINK HAPPENS IS
YOU DO THIS ADVANCED WORK WITH
THESE INCREDIBLY POWERFUL
MODELS, AND THEN WHEN IT COMES
TO FOLKS TAKING THESE THINGS TO
THE FDA, YOU GO BACK TO THE
MIDDLE AGES.
AND IF THESE THINGS CAN BE PUT
TO THE FDA, GET THEM HEAVILY
INVOLVED, HOW YOU'RE GOING TO
USE THIS, TO ACCELERATE CLINICAL
TRIALS, I THINK THAT WILL BE
TREMENDOUSLY POWERFUL.
BUT RIGHT NOW IT'S NOT, BECAUSE
IT'S DOING ALL THIS STUFF BUT
THAT NEXT STEP TAKING IT TO THE
HUMAN TRIALS IS REALLY MISSING
FROM THERE.
THE NEXT ONE IS, YOU MAY ALREADY
BE VERY WELL AWARE OF THIS, THIS
MAY BE REDUNDANT COMMENT, BUT I
KNOW FROM RARE DISEASE CLINICAL
RESEARCH AND OTHER GROUPS, ATCC,
CORIELL, HAVE VAST REPOSITORIES
OF RARE DISEASE CELL LINES THAT
REFLECT AN INSANE LEVEL OF
DIFFERENT TYPES OF PHYSIOLOGY.
THAT LEADS TO MY COMMENT, WHICH
IS IN THESE TISSUE MODELS,
SIGNALS ARE OFTEN BLUNTED WHEN
YOU'RE USING WHAT I WOULD SAY
TISSUE FROM THE MAINSTREAM
POPULATION, WHEREAS IF YOU CAN
FIND A RELEVANT RARE DISEASE
MODEL, THOSE EXTREME SYSTEMS
WILL OFTEN GIVE A SIGNAL THAT
YOU CAN THEN VALIDATE INTO MORE
GENERAL POPULATION BUT YOU MIGHT
OTHERWISE MISS, SO I WOULD
STRONGLY ENCOURAGE YOU AS MUCH
AS POSSIBLE TO DEVELOP A LOT OF
THOSE RARE DISEASE MODELS, FOR
THE BENEFIT OF RARE DISEASE BUT
FROM THE STANDPOINT OF WHAT THEY
CAN TEACH YOU AND HOW THEY MAY
REACT IN NORMAL PHYSIOLOGY
RESPONSES.
BUT KEEP IT UP.
>> POINTS WELL TAKEN.
TISSUE CHIPS ARE PART OF THE I3D
RARE, AS I'VE MENTIONED IN MY
TALK, CLOSE TO OVER, THE EASE OF
USING TISSUE CHIPS IN TERMS OF
PATIENT DERIVED MATERIALS ARE
THE BIG ADVANTAGE ESPECIALLY THE
RARE DISEASE POPULATION.
THAT'S WHY I GAVE THE EXAMPLE OF
RARE NEUROPATHIES,
STRAIGHTFORWARD, ALL IT TOOK WAS
SIX MONTHS OF WORK, IT WENT
RIGHT INTO CLINICAL TRIALS.
>> YEAH.
I'VE GOT TO TELL YOU THE FDA IS
NOT AS AWARE AS THEY NEED TO BE.
THEY CAN BE -- ARE THERE ANY FDA
PLEBS MEMBERS ON THE CALL?
I DON'T THINK SO.
THEY CAN BE REACTIONARY.
>> FDA HAS BEEN A PARTNER IN THE
DEVELOPMENT OF THIS PROGRAM.
THEY HAVE PROVIDED REGULATORY
ADVICE, AND AS I MENTIONED IN
THE TRANSLATIONAL CENTERS FOR
MPS WHERE WE'RE ACTUALLY
KNOCKING AT THE DOOR IN TERMS OF
REGULATORY QUALIFICATION THEY
WILL BE INTIMATELY INVOLVED, AND
WE'RE WORKING ALSO WITH C-PATH,
NON-PROFIT ARM OF FDA.
>> I KNOW C-PATH WELL, YES.
DEFINITELY AROUND THE
QUALIFICATION PROGRAM BECAUSE
THAT WAS THE WAY WE GOT DRUG
DEVELOPMENT COMPANIES TO LOOK AT
THIS TOO.
IT'S GOT AN OFFICIAL "THIS IS
OKAY TO USE" KIND OF THING.
>> CORRECT.
>> GREAT.
THANK YOU.
KELLY?
>> I'M OFF MUTE.
THAT WAS AWESOME.
SECOND, AT SOME POINT IT MIGHT
BE USEFUL TO SHARE WITH YOU AN
OPEN SOURCE BIOLOGICAL DIGITAL
TWIN PROGRAM WE'VE BEEN HELPING
A COUPLE RARE DISEASE
ORGANIZATIONS START THAT ARE
FOCUSED ON AUTOIMMUNE
POPULATIONS, AND SOME RARE
AUTOIMMUNE POPULATIONS, WITH A
LOT OF TISSUE COLLECTION, SO
THAT MAY BE USEFUL AT SOME
POINT.
AS A BRASS TACKS MATTER I WANT
TO MAKE SURE YOU HAVE WHAT YOU
NEED AT AEROSPACE COMPANIES, IN
YOU NEED HELP NORTHROP GRUMMAN
OR LOCKHEED, LET ME KNOW.
>> THANK YOU.
>> WE'D APPRECIATE THAT.
ANY OTHER QUESTIONS FOR DAN?
OKAY.
DAN, THANK YOU SO MUCH AS ALWAYS
FOR YOUR GREAT PRESENTATION.
AND WE HAVE ONE MORE TO GO, I'M
DELIGHTED TO INTRODUCE OUR NEW
DIRECTOR FOR OFFICE OF STRATEGIC
ALLIANCES, I HAVE ASKED HIM TO
FOCUS ON HIS VISION FOR OSA, AND
LET YOU KNOW MORE ABOUT WHAT
HE'S BEEN DOING ABOUT AND UP TO
AND GET YOUR INPUT.
BALKI, OVER TO YOU.
>> GOOD AFTERNOON.
THIS HAS BEEN A WONDERFUL DAY SO
FAR.
WE'VE HEARD GREAT SCIENTIFIC
TALKS, AS WELL AS VISIONARY
TALKS.
I'LL ADD SPIN BY POSITING THAT
TRANSLATION IS NOT JUST
SCIENTIFIC SCIENCE BUT ALSO A
LEGAL CHALLENGE AND, YOU KNOW,
BUSINESS CHALLENGE.
SO I'M THE DIRECTOR OF OFFICE OF
STRATEGIC ALLIANCES, LIKE JONI
MENTIONED.
WHAT I'LL DO TODAY IS PROVIDE A
PROGRAM UPDATE AND ALSO SHARE MY
VISION FOR THE OFFICE.
A LITTLE BIT OFF FREE-WHEELING
THINKING, VISUALIZING IZING KIND
OF SESSION.
NEXT SLIDE PLEASE.
I WANT TO START WITH THIS
PARTICULAR GRAPHIC THAT IS
POPULAR.
THIS SHOWS THAT NCATS FIRST USED
MISSION BY REENGINEERING
TRANSLATIONAL PIPELINE FOR
EFFICIENCY AND SEED.
THE OFFICE OF STRATEGIC ALLIANCE
WE SUPPORT ALL NCATS EFFORTS
EACH STEP OF THE WAY IN EACH OF
THE 3-Ds.
BY WAY OF EXAMPLES AND
DESCRIBING SOME OF THE TOOLS WE
USE, I'LL TELL YOU HOW OSA'S
WORK IS CENTRAL AND CRITICAL FOR
NCATS SUCCESS..
IT SUPPORTS TRANSLATIONAL
MISSION BY COMBINING SCIENCE,
LAW, BUSINESS INTO ONE USABLE
PRODUCT.
ON A PRACTICAL LEVEL, IN ORDER
TO REALLY DO TRANSLATIONAL
SCIENCE, ON TOP OF GREAT SCIENCE
WE NEED TO INFUSE BEST ELEMENTS
OF BUSINESS AND LAW TO MAKE
TRANSLATION TRULY SUCCESSFUL.
AND IN TERMS OF OUR OFFICE,
THERE ARE THREE DIFFERENT
FUNCTIONS THAT WE SUPPORT,
INTELLECTUAL PROPERTY
MANAGEMENT, ALLIANCE
MANAGEMENTS, SMALL BUSINESS
PROGRAMS.
I'M FOCUS TODAY ON INTELLECTUAL
PROPERTY MANAGEMENTS AND
ALLIANCE MANAGEMENT, I'LL TALK
ABOUT SMALL BUSINESS PROGRAMS
NEXT YEAR.
NEXT SLIDE PLEASE.
SO THROUGH THIS SLIDE, THE NEXT
FEW SLIDES, I WANT TO FOCUS MORE
ON WHAT I CALL OSA VISION.
JUST LIKE THE NCATS VISION, THE
OSA VISION IS FAIRLY SIMPLE, A
SIMPLE SENTENCE.
TO MAKE NCATS THE PREFERRED
PARTNER, AIMS TO MAKE NCATS THE
PREFERRED PARTNER FOR
COLLABORATING ON TRANSLATIONAL
PROJECTS BY INCREASING
EFFICIENCIES AND BY REDUCING
BARRIERS.
SO LET ME SORT OF GIVE YOU A
LITTLE BIT OF A SCIENTIFIC
ANALOGY, IN TERMS OF WHAT A
PREFERRED PARTNER WOULD BE.
WHEN YOU THINK OF CHEMISTRY
WORLD, THINK OF THE CARBON ATOM,
IT HAS UNIQUE PROPERTY OF BEING
ABLE TO BOND WITH ATOMS MUCH
SMALLER THAN IT IS, LIKE
HYDROGEN, MUCH LARGER LIKE
SULPHUR AND CHLORINE, AND ATOMS
JUST LIKE ITSELF, CARBON,
NITROGEN, OXYGEN.
JUST LIKE CARBON IS ABLE TO BOND
WITH ALL SIZES AND SHAPES AND
KINDS OF OTHER ATOMS TO MAKE
WONDERFUL MOLECULES, WE WOULD
LIKE NCATS TO BE THE PREFERRED
PARTNER THAT OTHER
ORGANIZATIONS, SMALL, LARGE,
MEDIUM, WHEREVER THEY ARE, TO BE
ABLE TO BOND WITH US, TO BE ABLE
TO PARTNER WITH US AND TAKE
TRANSLATIONAL PROGRESS FORWARD.
NOW, THE NCATS VISION IF IT
IS -- OSA VISION, ONCE IT IS
COMPLETELY PUT INTO PRACTICE,
WILL SUPPORT EACH ELEMENT OF
NCATS' VISION.
MORE TREATMENTS BY INNOVATING,
BY HAVING A VOLUME AND SPEED OF
NEGOTIATIONS FAIRLY SUPERIOR,
PROVIDE MORE SLOTS ON GOAL,
LEADING TO MORE TREATMENTS.
BY DOING AGREEMENTS WITH SPEED
AND EFFICIENCY, BY USING
TEMPLATES, I'LL GIVE EXAMPLES,
BUILDING NETWORKS AND
RELATIONSHIPS WILL HAVE
RELATIONSHIPS AND THE DISCOVERY
EVER DRUGS GO MORE QUICKLY, BY
USING PLATFORM TECHNOLOGIES,
REDUCING BARRIERS TO
COLLABORATIONS WE CAN INCLUDE
ALL PEOPLE IN THE PICTURE.
WHAT DOES IT TAKE TO BE A
PREFERRED PARTNER?
THERE ARE AT LEAST SIX ELEMENTS,
MAYBE MORE, THAT ARE SHOWN IN
THIS SCHEMATIC PLANETARY
DIAGRAM.
THE FIRST ONE THAT COMES TO MIND
IS TRUST.
TRUST IS SOMETHING THAT EVERY
SPEAKER IN THIS PANEL TODAY AS
WELL AS OTHERS HAVE SPOKEN ABOUT
TRUST IS SO IMPORTANT IN
COLLABORATIONS, IN SCIENCE, IN
PRETTY MUCH ANYTHING YOU DO
WHERE YOU WANT TO MAKE PROGRESS.
TRUST IS SOMETHING THAT'S
EARNED, DEPENDS ON SOME SORT OF
TRACK RECORD, SO BY HAVING A
LARGE VOLUME OF COLLABORATIONS
DONE IN VERY SMALL MANNER, YOU
CAN EARN TRUST.
SPEED IS ALSO IMPORTANT WHEN YOU
HAVE PARTNERS, PREFERENTIALLY
WORKING WITH YOU.
THEY WANT TO BE ABLE TO GET
THINGS DONE QUICKLY.
INNOVATION IS ANOTHER THING THAT
PARTNERS LOOK FOR IN A PARTNER.
I'M GOING TO GO THROUGH SOME OF
THE CASE STUDIES AND EXAMPLES
AND WE'LL COME BACK TO THIS AT
THE VERY END SO WE CAN TALK
ABOUT HOW WE CAN MAKE OURSELVES
EVEN STRONGER, TO MAKE OSA THE
PREFERRED PARTNER.
IN THE NEXT SLIDE, NEXT SLIDE
PLEASE, SO THIS IS THE OFFICE OF
STRATEGIC ALLIANCES, I'M VERY
FORTUNATE TO HAVE A GREAT TEAM
OF VERY DIVERSE, CREATIVE, AND
DEDICATED INDIVIDUALS.
THESE ARE INDIVIDUALS WHO USE
DIFFERENT ASPECTS OF SCIENCE OR
BUSINESS OR LAW, DIFFERENT
COMBINATIONS, JUST LIKE THE
CARBON ATOM.
IT USEST ORBITAL TO BOND WITH
DIFFERENT ATOMS, DEPENDING ON
THE ATOM MIGHT USE DIFFERENT
PROPERTIES, JUST LIKE THAT MANY
OF THE PEOPLE IN MY STAFF, THEY
USE DIFFERENT SKILLS AS WELL AS
PERSONALITY TYPES, AS WELL AS
BACKGROUNDS TO FORM THE RIGHT
PARTNERSHIPS.
I'LL TALK ABOUT SOME OF THE WORK
TODAY BUT WHAT I WANT YOU TO
TAKE AWAY FROM THIS CONVERSATION
IS THE FACT THAT IN ORDER TO
REALLY FORGE PARTNERSHIPS YOU
HAVE MULTIPLE ELEMENTS COMING
INTO PLAY.
TRUST, FLEXIBILITY, SPEED,
EFFICIENCY, ET CETERA.
NEXT SLIDE.
I WANT TO SHOW YOU IN THIS SLIDE
AND A COUPLE OTHER SLIDES SOME
OF THE STATISTICS OF OUR OFFICE.
SO IN THIS PARTICULAR SLIDE I'M
GIVEN YOU A SNAPSHOT OF THE
NUMBER OF AGREEMENTS THAT NCATS
HAS DONE OVER THE LAST TWO
YEARS, FY 22 AND FY 23.
I'LL GO QUICKLY, BUT ALSO DWELL
MORE ON OTHERS.
RIGHT ON TOP IS COOPERATIVE
RESEARCH AND DEVELOPMENT
AGREEMENT, CRADA, SOME OF YOU
ARE FAMILIAR WITH.
THESE ARE FAIRLY COMPLICATED
AGREEMENTS THAT TAKE SEVERAL
MONTHS, SOMETIMES EVEN YEARS TO
NEGOTIATE, IT HAS A LOT OF BELLS
AND WHISTLES, IT CAN BRING MONEY
IN, BRING RESOURCES IN, IT HAS A
LOT OF CLAUSES THAT DEAL WITH
INTELLECTUAL PROPERTY, SOMEWHAT
COMPLICATED TO NEGOTIATE.
WE DID ABOUT FOUR IN THE LAST
TWO YEARS.
NIH TYPICALLY DOES ABOUT 40 OR
50 A YEAR, SO IN THAT SENSE IT'S
IS A FAIRLY DIFFICULT AGREEMENT
NEGOTIATE, ALSO VERY FAR
REACHING.
MATERIAL TRANSFER AGREEMENTS ARE
STRAIGHTFORWARD, FOUR TO FIVE
PAGES LONG IN MTAs, 200 A
YEAR, RESEARCH COLLABORATION
AGREEMENT IS PROBABLY THE MOST
ROBUST, MOST EFFICIENT
AGREEMENT.
I CALL IT THE WORKHORSE
AGREEMENT WITH HIGHEST
EFFICIENCY.
IT'S AN AGREEMENT THAT CAN
ACTUALLY BE PUT INTO PLACE
WITHIN DAYS OR WEEKS, ABOUT FIVE
TO EIGHT PAGES LONG,
INCORPORATION THE RESEARCH PLAN,
LOTS OF CONDITIONS IN TERMS OF
PUBLICATION RIGHTS, I.T. RIGHTS,
CONFIDENTIAL CLAUSES, INCLUDES A
LOT OF IMPORTANT CLAUSES IN AN
EFFICIENT WAY.
I WALL THIS ONE AGREEMENT, IF
SOMEBODY WERE TO TELL ME THERE'S
ONLY ONE YOU COULD USE, I WOULD
CHOOSE THIS ONE.
IT DOES A LOT AND IS ALSO VERY
EFFICIENT.
WE DO A NUMBER OF OTHER KINDS OF
AGREEMENTS, THE REASON WE HAVE
SO MANY DIFFERENT AGREEMENTS IN
PLACE THAT WE USE IS BECAUSE
EACH COLLABORATION IS VERY
DIFFERENT.
SOME MAY LAST A FEW DAYS OR
WEEKS, OR MONTHS, OTHERS MAY
LAST FOR YEARS, MAYBE A DECADE.
, DEPENDING ON THE AGREEMENT,
THE PARTNER, WHAT YOU WANT TO
ACHIEVE YOU USE DIFFERENT KINDS
OF AGREEMENTS TO BEAR ON THE
PROBLEM.
THE NEXT SLIDE, I WANT TO TALK
ABOUT THE INTELLECTUAL PROPERTY
MANAGEMENT ASPECT OF NCATS.
SO WHAT I'VE SHOWN █HERE, GOING
FROM LEFT TO RIGHT, ARE EIGHT
DIFFERENT INSTITUTES WITHIN NIH,
TWO SMALL INCLUDING NCATS, FOUR
MEDIUM, AND TWO LARGE
INSTITUTES.
WHAT YOU CAN SEE ON THE TOP OF
THE TABLE IS THE NUMBER OF
INTELLECTUAL PROPERTY FILINGS
DONE OVER THE LAST TWO YEARS BY
THESE INSTITUTES.
NCATS 62 IN THE LAST TWO YEARS,
INTELLECTUAL PROPERTY FILINGS.
WHAT I'VE SHOWN IN THE LINE
BELOW THAT IS THE BUDGET, THE
INTRAMURAL RESEARCH BUDGET FOR
EACH OF THE INSTITUTES.
GOING FROM $261 MILLION TO $2
BILLION ON THE RIGHT.
WHAT I THEN DO IS I DIVIDE FIRST
NUMBER BY SECOND NUMBER IN ORDER
TO NORMALIZE AMOUNT OF
INTELLECTUAL PROPERTY GENERATED
PER UNIT OF CURRENCY, LAST LINE.
WHAT YOU SEE IN THE LAST LINE IS
WHAT I WOULD CALL INTELLECTUAL
PROPERTY ACTIVITY FOR $100
MILLION.
THE LARGER THE NUMBER, THE MORE
I.P. IS GENERATED FOR A GIVEN
DOLLAR.
BOTTOM LINE, NCATS GENERATES
MORE INTELLECTUAL PROPERTY WITH
FEWER DOLLARS, WE COMPARE WELL
WITH EVEN SOME LARGER
INSTITUTES.
NOW, THIS IS SOMETHING TO BE
PROUD OF, AND VERY NOTABLE.
WHAT COULD BE THE REASON FOR
SOME OF THIS?
THERE ARE A NUMBER OF REASONS
LIKE THE FACT THAT NCATS, LIKE
MANY THAT HAVE SPOKEN BEFORE ME,
USES COLLABORATIONS TO EXPAND
ITS SCIENTIFIC REACH.
WE ALSO FOCUS ON PLATFORM
TECHNOLOGIES.
WE EMPLOY TEAM SCIENCE TO DERIVE
GREATER SYNERGY AND IDEATION.
SO ALL THESE THINGS PROBABLY
HELP A LOT IN MAKING SURE THAT
WE USE THE BEST -- MAKE BEST USE
OF RESEARCH DOLLARS GIVEN TO US.
FINALLY, THE CLOSE WORKING
RELATIONSHIP BETWEEN THE OFFICE
OF STRATEGIC ALLIANCES AND
SCIENTISTS PLAY A HUGE ROLE IN
THE SUCCESS.
WE CAN USE THE BEST COMBINATION
OF BUSINESS, SCIENCE, LAW, TO
FORM THE RIGHT PARTNERSHIPS,
ACHIEVE THE RIGHT TRANSLATIONAL
RESULTS.
I THINK ALL OF THAT COMES
TOGETHER IN WHAT IS NCATS.
SO, WHEN YOU LOOK AT A TABLE
LIKE THIS IT GIVES YOU AN IDEA
THAT NCATS IS HAS HIT ON UNIQUE
WAYS TO INCREASE PACE OF
TRANSLATION, SOMETHING WE CAN
ALL BE VERY PROUD OF.
THE NEXT SLIDE, I SHOW A LITTLE
BIT ABOUT THE LICENSES THAT
NCATS HAS DONE OVER THE LAST 15
YEARS.
LET ME PAUSE FOR A MINUTE AND
SAY THAT NCATS, PART OF NIH,
PART OF THE FEDERAL GOVERNMENT,
WE CANNOT COMMERCIALIZE
PRODUCTS.
SO LICENSING IS A CONVENTIONAL
OUTCOME OF INVENTIONS WE CREATE.
WE LOOK FOR PARTNERS ON THE
OUTSIDE, COMMERCIAL SECTOR, TO
TAKE THEM AND BUILD THEM INTO
PRODUCTS.
I'LL GIVE SOME EXAMPLES OF HOW
WE'VE DONE THAT.
SO, COMMERCIAL LICENSES, PATENT
COMMERCIAL LICENSES, WE'VE DONE
EIGHT, SOME HAVE BEEN VERY FAR
REACHING.
IN THE MIDDLE OF THE TABLE I
SHOW IIA, THESE ARE AGREEMENTS
BETWEEN TWO INSTITUTIONS THAT
CO-OWN A PIECE OF INTELLECTUAL
PROPERTY.
WHEN YOU COLLABORATE WITH
ANOTHER INSURE INSTITUTION,
JOINT INVENTION, IT'S PREFERABLE
TO GET AN AGREEMENT THAT SAYS
ONE OR THE OTHER ORGANIZATION
WOULD TAKE THE LEAD ON THE IP,
MANAGING IP, MARKETING IP,
PERHAPS FINDING LICENSES PARTNER
DURING NEGOTIATIONS.
IN THE CASE OF NCATS, BECAUSE OF
COLLABORATIVE NATURE, MANY MORE
OF OUR IIAs, INSTITUTIONAL
AGREEMENTS, HAVE THE OUTSIDE
INSTITUTION TAKING THE LEAD AND
NIH TAKES THE LEAD ON A SMALLER
PROPORTION OF THOSE.
I'LL GIVE EXAMPLES OF EACH OF
THOSE CATEGORIES OF IIAs.
NEXT SLIDE PLEASE.
SO I'M GOING TO SWITCH GEARS AND
PRESENT SOME CASE STUDIES TO
EXEMPLIFY HOW OSA HAS BEEN DOING
ITS WORK IN COLLABORATION WITH
THE SCIENTISTS, IN COLLABORATION
WITH OUTSIDE PARTNERS.
NEXT SLIDE PLEASE.
AS YOU REMEMBER, THE VISION
CIRCLE WITH THE PLANETARY
DIAGRAM OF THE DIFFERENT
ELEMENTS, ONE OF THE ELEMENTS I
MENTIONED WAS INNOVATION.
SO THIS IS A STORY I WANT TO
TELL YOU IN A LITTLE BIT OF
DETAIL BECAUSE I WANT TO TELL
YOU ABOUT THE INNOVATION THAT
OUR OFFICE HAS DONE IN THE
COLLABORATION PROCESS ITSELF.
NOW, INNOVATION IS EASY TO SEE
IN SCIENCE, EASY TO SEE IN
ENGINEERING, IN FACT YOU CAN
ACTUALLY LOOK AT A PIECE OF
INNOVATION AND YOU CAN TOUCH IT,
FEEL IT, EVEN TASTE IT.
IN THE FIELD OF BUSINESS AND
LAW, WHEN THERE ARE INNOVATIONS
THEY ARE FEW AND FAR IN BETWEEN.
THEY ARE VERY HARD TO REALLY
UNDERSTAND AND EVEN EXPLAIN.
SO I'M GOING TO TAKE A FEW
MINUTES TO EXPLAIN WHAT THIS
INNOVATION REALLY IS.
REMEMBER I TALKED ABOUT THE
CRADA, THE CRADA AGREEMENT IS
SOMETHING THAT WAS WRITTEN IN
THE 1980s, ALONG WITH
TECHNOLOGY TRANSFER LAWS, AND
HAS BEEN IN EXISTENCE FOR OVER
40 YEARS.
TALK ABOUT INNOVATION, THERE WAS
ONE INNOVATION IN THE CRADA
AGREEMENT WHICH WAS THE
MATERIALS CRADA IN THE EARLY
2000s, WHEN COMPANY WANTED TO
PROVIDE MATERIALS TO NIH, WANTED
INTELLECTUAL PRODUCT RIGHTS, THE
MATERIALS CRADA.
IN 2020, NCATS DEVELOPED A NEW
KIND OF CRADA CALLED C-RCA, WHAT
IS THE IMPETUS AND MOTIVATION
FOR THIS?
THE RCA LIKE I MENTIONED IS
PROBABLY THE MOST EFFICIENT OF
THE COLLABORATION AGREEMENTS.
THE CRADA IS SOMEWHAT
COMPLICATED.
IT'S GOT A LOT OF LEGAL
PROTECTIONS.
AND LIKE GOLDILOCKS, OSA WAS
LOOKING FOR SOMETHING JUST
RIGHT, WHICH WOULD COMBINE THE
LEGAL CRADA, WITH SPEED AND
EFFICIENCY.
WE CREATED C-RCA, COMBINING THE
BEST ELEMENTS.
WE'RE USED IT 25 TIMES, OTHER
INSTITUTES STARTED USING IT.
BENEFITS OF C-RCA, IT TAKES
WEEKS TO MAYBE DAYS TO COMPLETE
VERSUS MONTHS FOR A CRADA TO
COMPLETE BUT HAS THE SAME LEGAL
COVERAGE.
LIKE I SAID, PRESERVES LEGAL
SAFEGUARD FOR BACKGROUND IP,
IT'S BEEN USED OVER 25 TIMES AT
NCATS, WE'VE PUBLISHED ON IT SO
WE CAN DISSEMINATE TO LARGER
COMMUNITY.
NEXT SLIDE PLEASE.
SO ANOTHER EXAMPLE I WANT TO
SHOW YOU IS IN THE AREA OF
EFFICIENCIES.
WHEN YOU HAVE A SITUATION LIKE
THE N3C WHEN THE N3C PROJECT
FISHES STARTED WE WANTED TO
INGEST DATA FROM MANY, MANY
DIFFERENT INSTITUTIONS.
WE WANTED LITERALLY TO HAVE A
SITUATION WHERE THINGS COULD BE
DONE IN A VERY STREAMLINED
FASHION, AND WE ALSO ARE ABLE TO
SCALE THINGS UP.
IMAGINE IF 20 TO 30 PARTNERS
COME TO YOU AND SAY WE WANT TO
FILE AN AGREEMENT TO DO THIS.
THERE'S NO EASY SOLUTION.
WHAT WE DECIDED TO DO, CHRIS
DILLON TOOK ON THIS CHALLENGE,
SAID WE NEED TO COME UP WITH
SOME TEMPLATE AGREEMENT THAT
WILL BE USABLE, ROBUST, AND
PALATABLE TO A LOT OF PEOPLE AND
DEVELOPED WITH LILLY PORTILLO,
THREE AGREEMENTS, DTA, DUA,
PPRL, FOUR MONTHS TO GENERATE IN
TERMS OF ROBUST STRONG TEMPLATE.
IN DEVELOPING THESE TEMPLATES,
MULTIPLE PERSPECTIVES WERE
TAKEN, IN ADDITION TO THE
POTENTIAL DATA USERS, AND DATA
PROVIDERS.
SO, BY USING THIS PARTICULAR
TEMPLATE AND POWER OF
FLEXIBILITY OF TEMPLATE WE WERE
ABLE TO EXECUTE THE NC 3 DATA
USE AND DATA TRANSFER AGREEMENTS
OVER 500 TIMES, AND HAD TO MAKE
ADJUSTMENTS ONCE OR TWICE,
FAIRLY MINOR CHANGES.
SO SPEED AND EFFICIENCY IS
SOMETHING WE ARE VERY PROUD OF
IN TERMS OF WHAT WE DO AT NCATS,
OFFICE OF STRATEGIC ALLIANCES.
THE NEXT SLIDE PLEASE.
SO, THIS SLIDE I WANT TO GIVE AN
EXAMPLE OF HOW WHEN YOU DEVELOP
A PLATFORM TECHNOLOGY, WHAT'S
THE BEST WAY TO DISSEMINATE IT?
SO THIS IS A STORY THAT WAS --
DESCRIBES THE WORK DONE AT THE
STEM CELL RESEARCH LAB LED BY
CARLOS AND HIS COLLEAGUES
VERY CHALLENGING MOST PEOPLE WHO
WORK WITH STEM CELLS KNOW THIS.
GROWING, CARING FOR, EXPANDING
STEM CELLS IS NOT A JOKE.
THE PROCESSES ARE DIFFICULT,
PEOPLE USE ALL KINDS OF GROWTH
FACTORS, BUT IN THIS PARTICULAR
CASE THE STEM CELL RESEARCH
LABORATORY REVOLUTIONIZED THE
FIELD BY COMING UP WITH THE
FOUR-PART COCKTAIL OF SMALL
MOLECULES, WELL DEFINED, FOUR
MOLECULES, WHICH PUT TOGETHER
CAN MAKE STEM CELL PROCESS MUCH,
MUCH MORE EASY AND STREAMLINED.
BY USING THE CEPT FOUR-PART
COCKTAIL OF WELL-DEFINED
MOLECULES, BY THE WAY TOOK
SEVERAL MONTHS TO DEVELOP THIS
BY HIGH-THROUGHPUT SCREENING AND
ASSAYS, I WON'T GO INTO THAT,
BUT THE PARTICULAR COCKTAIL WAS
ABLE TO INCREASE CELL VIABILITY,
REDUCE DNA DAMAGE, CREATE
PHENOTYPIC ACCURACY IN TERMS OF
CELL LINES GROWING, AND SUPPORTS
FUNCTION OF MATURE CELLS.
NEXT SLIDE.
SO, ONCE THIS INVENTION CAME TO
OUR OFFICE, LOOKED AT SAID MY
GOD, THIS IS REVOLUTIONARY
PLATFORM, NIGHT TO GET IT TO THE
MOST NUMBER OF USERS AS QUICKLY
AS POSSIBLE.
SO HE TOOK AN APPROACH THAT
WOULD BE CALLED NON-EXCLUSIVE
LICENSING, ON NON-EXCLUSIVE
BASIS, TRY TO COMMERCIALIZE IT.
WHILE DOING THIS, TOOK FOUR
APPROACHES.
FIRST WAS INTERNAL ALLS
NON-EXCLUSIVE LICENSE WHERE HE
WOULD LICENSE IT TO COMPANIES
FOR INTERNAL USE, EVEN OTHER
NON-PROFIT AGENCIES.
THERE WERE FIELD SPECIFIC
COMMERCIAL NON-EXCLUSIVE LICENSE
WHERE PATENT WAS LICENSED ON
NON-EXCLUSIVE BASIS FOR CERTAIN
FIELDS.
THERE WAS ALSO COMMERCIAL
EVALUATION LICENSES SO A COMPANY
THAT WANTS TO COMMERCIALIZE IT
COULD TAKE IT FOR A SMALL PERIOD
OF TIME, EVALUATE US, TELL US
WHAT THEY WANT TO DO AFTER THAT.
FOR THE ACADEMIC, WE'LL GIVE A
MATERIAL TRANSFER AGREEMENT THAT
WILL ALLOW YOU ACCESS TO THE
METHODOLOGY AND MATERIALS ON A
SHORT-TERM BASIS.
SO BOTTOM LINE NON-EXCLUSIVELY
LICENSING TO A NUMBER OF
DIFFERENT COMPANIES, THE
COMPANIES LISTED HERE, HE WAS
ABLE TO REACH THE -- PROVIDE
STEM CELL COCKTAIL PLATFORM THAT
HAS MANY DIFFERENT USERS WITH AS
MUCH REACH AND AS LITTLE PAIN AS
POSSIBLE.
NEXT SLIDE PLEASE.
SO THIS IS AN EXAMPLE OF A
LICENSE NEGOTIATION WHICH WAS
EXCLUSIVELY LICENSE WHERE THE
COLLABORATOR TOOK THE LEAD.
IN THIS CASE PRESENTED WITH
INVENTION, TREATMENT FOR RARE
CANCERS WITH SMALL MOLECULES.
ONE OF THE LABS LED BY CRAIG
THOMAS AND OTHERS AT NCATS WITH
THE CINCINNATI GROUP DEVELOPED
SMALL MOLECULES VERY PROMISING.
IN THOSE EARLY STAGES, AMY TOOK
A LOOK AND SAID, WHAT'S THE BEST
WAY TO GO FORWARD?
SHOULD NCATS OR NIH TAKE THE
LEAD OR CINCINNATI TAKE THE
LEAD?
BASED ON THE DATA AND ALL THE
ANALYSIS SHE CONCLUDED THAT THE
BEST THING WOULD BE FOR THE
CINCINNATI GROUP TO TAKE THE
LEAD, SO NEGOTIATED
INTERINSTITUTE AGREEMENT WITH
CINCINNATI CHILDREN'S HEALTH
MEDICAL CENTER, AND IIA
TYPICALLY AN EXCLUSIVE LICENSE
WHICH GIVERS THE OTHER PART FULL
RIGHTS TO FILE THE PATENTS AS
WELL AS MARKET THE PATENT AND
FINE LICENSEES.
NOW, AT THE END OF THE DAY THERE
WERE 86 PATENT APPLICATIONS,
WITH LOTS OF COMPLEXITY.
AMY MADE THE RIGHT CHOICE
BECAUSE MANAGING ALL THOSE
APPLICATIONS, FOOTING THE BILL,
TAKING ALL THE EXPENSES UP
FRONT WOULD HAVE BEEN A HUGE
UNDERTAKING.
SOME RISK AND BURDEN WAS SHIFTED
TO CINCINNATI.
SHE ALSO NEGOTIATED A THREE-WAY
CRADA WITH A START-UP COMPANY,
KURON, LICENSED THE TECHNOLOGY
FROM CINCINNATI.
NEXT SLIDE PLEASE.
IN TERMS OF COLLABORATION WITH
THE 3-WAY COLLABORATION
CINCINNATI SET ALL START-UP
COMPANY, KUROME, THAT GOT
FUNDING AND THEY ALSO WERE ABLE
TO PUT TOGETHER A BOARD AND A
NUMBER OF SCIENTISTS, SO THEY
COULD MANUFACTURE THE DRUG, DO
IND-ENABLING STUDIES, IND WILL
BE FILED PRETTY SOON.
THE NEXT SLIDE SHOWS JUST THE
OPPOSITE.
IN THIS PARTICULAR CASE, SORI
WAS GIVEN THE TECHNOLOGY WHICH
MANY OF YOU HEARD ABOUT, I WON'T
TALK ABOUT THE SCIENCE, BUT
BASICALLY FIRST IN CLASS
MOLECULE, METARRESTIN, ARRESTING
METASTASIS, FIRST IN CLASS
CANCER DRUG.
IN THIS CASE, HE DECIDED THE
BEST COURSE OF ACTION WOULD BE
FOR NCATS TO TAKE THE LEAD, WE
TOOK THE LEAD, FILED PATENT
APPLICATION IN COLLABORATION
WITH OTHER PARTNERS.
ALONG THE WAY THERE WAS SOME
FUNDING FROM THE THERAPEUTIC
DEVELOPMENT BRANCH TO DO
PRE-CLINICAL EVALUATION, SOME
IND-ENABLING WORK, AND SOME
PHASE 1 WORK WAS DONE IN NCI.
AND THEN BASED ON THAT, WE HAVE
A LICENSING PARTNER NOW, WHICH
CAN TAKE A PHASE 1 ASSET AND
DEVELOP IT EVEN FURTHER.
SO, I'VE SHOWN TWO DIFFERENT
WAYS OF APPROACHING THE SAME
PROBLEM.
WHEN YOU HAVE A JOINT INVENTION
YOU CAN TAKE THE LEAD YOURSELF,
ALLOW THE COLLABORATING PARTNER
TO TAKE THE LEAD.
EACH ONE HAS GOT PROS AND CONS,
YOU HAVE TO MAKE THE RIGHT
JUDGMENT AT THE RIGHT TIME.
NEXT SLIDE PLEASE.
SO THE LAST EXAMPLE OF THE LAST
CASE STUDY I WANT TO TALK ABOUT
IS SOMETHING THAT CHRIS DILLON
LED.
TRANSLATION IS BOTH SCIENTIFIC
AND BUSINESS CHALLENGE.
AND IN ORDER FOR A GOOD
TRANSLATIONAL SCIENTIST THEY
NEED THE RIGHT TOOLS OF SCIENCE
AND RIGHT BUSINESS TOOLS TO BE
ABLE TO BE A TRANSLATIONAL
SCIENTIST.
WE DEVELOPED A PROGRAM,
ADVANCING INNOVATION THROUGH
MENTORSHIP, PATENTED AFTER
I-CORPS HOW TO FIND OUT WHAT
POTENTIALS ARE, BASICALLY
EXPERIENTIAL LEARNING PROCESS
THROUGH CUSTOMER DISCOVERY
METHODOLOGY.
YOU USE THE WORD CUSTOMER IN A
BROAD WAY.
A CUSTOMER COULD BE POTENTIAL
COLLABORATOR, POTENTIAL USER,
COULD BE THOUGHT LEADER, COULD
BE A COMPETITOR, COULD BE
ANYBODY.
GROUPS ARE FORMED WHERE A GROUP
OF THREE OR FOUR PEOPLE WOULD
GET TOGETHER AND START DOING
INTERVIEWS OF MULTIPLE PEOPLE ON
THE OUTSIDE, PEOPLE WHO EITHER
KNOW ABOUT THE SUBJECT, ARE
EXPERTS, POTENTIAL USERS.
SO 14 TEAMS STRAINED OVER THE
LAST FOUR YEARS, 39 STAFF
TRAINED, SOME PROJECTS HAVE BEEN
VERY FASCINATING.
SO AT THE BOTTOM I LISTED THREE.
ONE LED BY BILL SANDERS, WORKING
ON THE BUNYAVIRUS RESEARCH,
DECIDED THEY WANTED TO FIND OUT
WHAT IS KEEPING THE FIELD
DORMANT, WHAT ARE SOME
CHALLENGES.
SO THEY STARTED TALKING TO
COLLABORATORS AND FOUND OUT
MERCK COULD BE POTENTIAL
COLLABORATOR.
THE OTHER TEAM WORKING ON A
METHODOLOGY, ENDED UP FILING
PATENTS, DEVELOPED PROTOTYPE
INSTRUMENT.
NEXT SLIDE PLEASE.
I WANT TO STOP ON THIS SLIDE AND
TALK ABOUT DIFFERENT ELEMENTS
INVOLVED, HOPEFULLY I'VE SHOWN
ALONG WITH CASE STUDIES, TRUST
IS MOST IMPORTANT, EARNED WITH
COLLABORATION AND PARTNERSHIPS.
WITH SPEED AND EFFICIENCILY THEY
WILL COME BACK.
IF YOU HAVE INNOVATIVE METHODS
TO REDUCE VISCOSITY, PARTNERS
COME.
SHOW FLEXIBILITY, THEY WILL
COME.
IF YOU ARE A BRIDGE BUILDER,
THEY WILL ALSO COME TO YOU.
ONE BIG CHALLENGE WHICH ANY
ORGANIZATION HAS IN DEVELOPING
PARTNERSHIPS IS TO DEVELOP
NETWORKS.
IN ORDER TO HAVE LOTS AND LOTS
OF PARTNERS AND TO BE THE
PREFERRED PARTNER YOU NEED TO
HAVE A BROAD AND DEEP NETWORK.
AND THAT'S ONE AREA WHERE I
THINK WE REALLY COULD DO A LOT
MORE.
I'M GOING TO STOP THERE AND ASK
THE COUNCIL FOR SUGGESTIONS,
QUESTIONS, FEEDBACK, ON ANY OF
THESE ELEMENTS OR ANYTHING THAT
I'VE TALKED ABOUT.
>> THANK YOU, BARKI.
I LIKE THE METAPHOR WITH
CHEMICAL ENTITY, THE MOLECULES,
TO HOW WE THINK ABOUT
COLLABORATIONS.
>> SERGIO, YOU'RE FIRST.
>> THANK YOU, BALKI.
ONE COMMENT I WANT TO MAKE IS
THAT IT'S JUST GREAT THAT YOU
SHARED WITH US THE VISION
ELEMENTS, AND YOU SPOKE ABOUT
EACH OF THEM, ESPECIALLY TRUST,
IN PARTICULAR TRUST.
TRUST IS FRONT AND CENTER IN ANY
OF THE ACTIVITIES, IT SEEMS TO
ME THAT YOU DO WITHIN OSA, AND I
HAVE BEEN THINKING, I WAS
THINKING THROUGHOUT YOUR
PRESENTATION, HOW CRITICAL IT
IS, I MEAN TRUST, IT IS FOR
DEVELOPING, DEMONSTRATING,
DISSEMINATING WHATEVER IS BEING
DONE.
THIS CUTS ACROSS ALL THE
PROGRAMS.
AND I WOULD ENCOURAGE YOU TO --
MAYBE YOU'RE ALREADY DOING IT.
YOU KNOW, YOU DEVELOP YOUR
PARTNERSHIPS.
AND DEVELOP THE PLANS.
THAT YOU REALLY THINK ABOUT
THOSE WHO STAND TO BENEFIT THE
MOST, FOR THIS PROGRAM, AND GET
THEM SYSTEMATICALLY INVOLVED IN
THOSE THREE CRITICAL PHASES.
THE THREE Ds.
I'M THINKING ABOUT IN PARTICULAR
DISSEMINATION.
IF YOU DO THAT, YOU WILL HAVE A
CADRE OR AUDIENCE THAT WILL
BECOME CHAMPIONS FOR WHAT YOU'RE
TRYING TO DISSEMINATE, I THINK.
JONI, I'M DELIGHTED, TRULY,
ACROSS THE VARIOUS PROGRAMS
TRUST HAS EMERGED AS A KEY
ELEMENT, YOU KNOW, AND I WOULD
ENCOURAGE ALSO NCATS TO MAKE IT
MORE KIND OF FRONT AND CENTER.
TRUST AND TRUSTWORTHINESS.
WE SPOKE ALREADY ABOUT
TRUSTWORTHINESS.
BECAUSE I THINK, YOU KNOW, ONE
OF THE KEY EXPERIENCES THAT WE
KEEP HAVING AS WE GO ABOUT DOING
TRANSLATIONAL RESEARCH, FOR
EXAMPLE, WITH ESPECIALLY
UNDERSERVED COMMUNITIES, A
TREMENDOUS DISTRUST THAT EXISTS
IN THOSE COMMUNITIES.
AND I WAS REALLY ELATED WHEN YOU
HOSTED THE BRIEF BIO BY MONICA
BERTAGNOLLI THAT SHE'S ALSO
FOCUSING ON RESTORING TRUST IN
SCIENCE, SOMETHING SO DAMAGED
YOU WE NEED TO ACTIVELY AND
COLLECTIVELY DO SOMETHING ABOUT
IT.
THANK YOU.
>> TRUST IS SOMETHING YOU HAVE
TO EARN, AND, YOU KNOW, IN MY
SIMPLE PHILOSOPHY TRUST IS
SOMETHING YOU CAN BUILD WITH A
GOOD TRACK RECORD.
ONE OF THE THINGS WE'VE SEEN AT
NCATS, THE MORE AGREEMENTS THAT
WE DO, THE MORE VOLUME THAT WE
DO, THE MORE TRUST WE BUILD.
AS WE WORK WITH PEOPLE, I TRY TO
TELL THE PEOPLE ON BOTH SIDES
THAT THE AGREEMENT ITSELF IS A
STARTING POINT.
AS YOU INTERACT WITH EACH OTHER
PLEASE BUILD AND MORE TRUST TO
CONTINUE TO HAVE MORE
COLLABORATION AND RELATIONSHIPS.
I AGREE, SERGIO, YOU HAVE TO
EARN AND KEEP EARNING.
>> RIGHT.
ONE LAST COMMENT.
I WAS AT THIS MEETING YESTERDAY
IN D.C., THE NATIONAL ACADEMY OF
MEDICINE, AND IT WAS A MEETING
ON POWER OF ENGAGEMENT,
SPECIFICALLY WITH
REPRESENTATIVES ACROSS THE
NATION, CDC AND MANY OTHER
ORGANIZATIONS, IT WAS SO
INSPIRING TO SEE THE ROLE COMING
FROM VARIOUS ANGLES, ONCE AGAIN
TRUST AND TRUSTWORTHINESS.
JONI, I'LL REACH OUT TO YOU AND
SHARE SOME OF THAT WORK BECAUSE
IT WAS INSPIRING.
>> I WOULD APPRECIATE THAT.
I'M SO GLAD, SERGIO, YOU'RE AT
THESE CONFERENCES AN MEETINGS TO
ALSO LEND YOUR VOICE AND TALK
ABOUT HOW IMPORTANT THIS IS TO
THE RESEARCH COMMUNITY, LET
ALONE THE ENGAGEMENT COMMUNITY
AND THE PATIENT COMMUNITY.
BUT I THINK WE ALL NEED TO HEAR
HOW IMPACTFUL THESE IDEAS OF
BUILDING TRUST ARE AND THEN AS
WE TALK ABOUT THEM MORE, THEY
WILL BECOME MORE IN OUR
INTEGRATED, INTO OUR EVERYDAY
APPROACHES.
THANK YOU FOR DOING THAT WORK.
PLEASE SEND ME THE MATERIALS.
I'D LOVE TO SEE THEM.
>> THANK YOU.
>> PAUL?
>> BALKI, GREAT PRESENTATION, I
APPRECIATED THE USE CASES, VERY
HELPFUL.
A COMMENT AND QUESTIONS.
I THINK WE ALL SEE OSA AS VITAL
FOR DISSEMINATION ESPECIALLY FOR
INTRAMURAL PROGRAM BUT
APPRECIATE USE CASE YOU GAVE
AROUND THE N3C PLATFORM AND
POWER AND ABSOLUTE NECESSITY FOR
STANDARD AGREEMENTS, TO DO THOSE
PROJECTS AT SCALE.
MY QUESTION REALLY IS AROUND THE
LICENSING NCATS AS A GOVERNMENT
ENTITY IS ALLOWED TO DO.
YOU MENTIONED SEVERAL USE CASES.
IS LICENSING FROM NCATS, IS IT
ALWAYS DONE AT NO COST TO THE
LICENSEE?
OR ARE THERE MODELS WHERE THERE
IS INFUSION OF INVESTMENT BACK
IN FOR FURTHER SHARING, ET
CETERA?
THIS IS JUST A NAIVE QUESTION
BUT I'VE ALWAYS BEEN CURIOUS.
>> IN BROAD TERMS SOME RULES AND
REGULATIONS GOVERN LICENSING,
LICENSING PRINCIPLES.
NIH CAN TAKE ROYALTY MONEY,
INTELLECTUAL PROPERTY THAT YOU
OUT-LICENSE, YOU CAN ESSENTIALLY
ASK FOR A COMMERCIAL DEVELOPMENT
PLAN, THE GROUP THAT WANTS TO
LICENSE THE TECHNOLOGY.
IF YOU LICENSE SOMETHING
NON-EXCLUSIVELY ROYALTIES AND
PAYMENTS ARE LOWER.
IF YOU LICENSE EXCLUSIVELY YOU
CAN PRETTY MUCH ASK FOR A FAIRLY
DETAILED COMMERCIAL DEVELOPMENT
PLAN AND BASED ON THAT, YOU CAN
BUILD IN MODELS OF PAYMENTS,
MILESTONE PAYMENTS, ROYALTY
PAYMENTS, UP.FRONT PAYMENTS.
EVERYTHING STARTS WITH
COMMERCIAL DEVELOPMENT PLAN
POTENTIAL LICENSEE SUBMITS TO
YOU.
YOU NEGOTIATE FROM THERE.
>> YOU'RE ALLOWED TO SET UP
ROYALTY PLANS, THAT CAN SEED THE
PROGRAM FOR OTHER DEVELOPMENT,
FURTHER DISSEMINATION?
>> YES, THE ROYALTY -- SOME
RULES IN TERMS OF HOW ROYALTIES
ARE DISTRIBUTED.
SOME PORTION OF THE ROYALTY GOES
TO INVENTORS, FROM THE PATENT,
THE REST GOES TO THE INSTITUTE.
>> THANK YOU.
>> THERE'S TYPICALLY A CAP TO
THAT AS WELL.
>> JUST ONE LAST QUESTION,
RESEARCH COLLABORATIVE
AGREEMENT, THE THE WORKHORSE,
HOW STANDARDIZED IS THAT
AGREEMENT?
SOUNDS LIKE YOU'RE DOING A TON
OF THOSE.
>> YES.
THOSE ARE FAIRLY STANDARDIZED
BUT THEY ARE A LOT MORE FLEXIBLE
SO SOMETIMES WHAT HAPPENS IS
DEPENDING ON THE RELATIVE VALUE
OF EACH PARTNER, EACH ONE GETS
TO NEGOTIATE, YOU KNOW, A LITTLE
BIT MORE OR LESS DEPENDING ON
WHO HAS THE UPPER HAND.
SO THERE'S SOME NEGOTIATION
INVOLVED BUT DEFINITELY THERE
ARE SOME LANGUAGE OR CLAUSES
FAIRLY STANDARD WHICH WE DON'T
CHANGE, BUT THERE ARE OTHERS
WHICH WE HAVE SOME FLEXIBILITY.
>> THANK YOU SO MUCH.
>> UH-HUH, SURE.
>> GREAT.
PAULA?
>> THAT WAS VERY IMPRESSIVE,
BALKI.
LET'S FACE IT, ALL OF US WHO DO
RESEARCH HAVE HAD TO SIT
TWIDDLING OUR THUMBS WHILE
CONTRACTS AND USE CASES AND ALL
OF THAT ARE BEING NEGOTIATED.
WE'VE ALSO TALKED EARLIER IN
THIS MEETING ABOUT HOW VALUABLE
PATIENT DATA IS AND ELECTRONIC
HEALTH RECORD DATA, SO N3C HAS
BEEN A GREAT EXAMPLE HOW HOW DO
YOU TAKE IT TO THE NEXT LEVEL,
HOW DO YOU WORK YOUR MAGIC TO
HAVE GREATER ACCESSIBILITY, EVEN
MEDICAL RECORDS TO A CLINICIAN,
WHO HAS PATIENTS GOING MULTIPLE
PLACES, BUT ALSO FOR THE
RESEARCH, THE MACHINE LEARNING,
THE LARGE LANGUAGE MODELS, ALL
THE THINGS THAT NEED THIS BIG
DATA, WHAT'S THE NEXT STEP IN
DATA SHARING THAT YOU AND NCATS
CAN KIND OF INNOVATE SO THAT
WE'RE NOT AT A STAND STILL SO
OFTEN?
>> YEAH, I THINK THAT'S A BIGGER
QUESTION THAN WHAT WE CAN
ANSWER.
BUT I THINK THERE ARE SOME WAYS
BY WHICH DATA CAN BE SHARED MORE
WIDELY.
IF SOMETHING DOESN'T HAVE TO BE
PATENTED MID A VISE IS PUBLISH
OF -- MY ADVICE IS PUBLISH IT,
SHARE IT WIDELY, SHARE THE
INFORMATION, SHARE THE
KNOW-HOUR.
BUT IF THERE ARE SITUATIONS
WHERE SOMETHING NEEDS TO BE
PATENTED, PATENT IT.
ONCE THE PATENT IS COMPLETED,
YOU CAN START DISSEMINATING
INFORMATION, A LOT OF TIMES THE
FEDERAL SCIENTISTS, THEY SHARE A
LOT OF KNOW-HOW WITH OTHER
PEOPLE, WITH OUTSIDE PEOPLE,
BECAUSE DATA BY ITSELF IS NOT
SUFFICIENT BECAUSE SOMETIMES
DATA HAS TO COME WITH
INTERPRETATION, WITH BACKGROUND,
CONTEXT, AND THAT'S WHERE I
THINK THE NIH SCIENTISTS REALLY
SHINE BECAUSE WE HAVE PROBABLY
ONE OF THE MOST OPEN DATA
SHARING PHILOSOPHIES, BUT,
AGAIN, IT IS SOMETHING THAT IS A
MUCH LARGER QUESTION AT THE NIH
LEVEL.
JONI, IF YOU WANT TO ADD
ANYTHING GO AHEAD.
>> IT'S A GREAT IDEA.
I THINK THAT IN MANY WAYS WE TRY
TO TEMPLATIZE THEM, THEN IT
COMES TO PERHAPS JUST MAKING
THOSE AVAILABLE IN A WAY.
AS BALKI TALKED ABOUT, N3C
AGREEMENTS, YOU KNOW, WE SPENT
THE BULK -- RATHER THAN GENERATE
AN AGREEMENT AND SENDING IT OUT
TO PEOPLE AND HAVE THEM CHANGE
IT WE SPENT THE BULK OF TIME
TALKING WITH PEOPLE FIRST,
UNDERSTANDING WHAT THE NEEDS
WERE, AND THEN BUILT THE
AGREEMENT THAT COULD ACCOMMODATE
EVERYTHING THAT WE NEEDED TO DO.
AND JUST PUTTING IN THAT LITTLE
WORK UP FRONT, YOU KNOW, SLOWING
DOWN UP FRONT HELPED US TO SPEED
THINGS UP SHORTLY THEREAFTER.
AND SO THAT SLOWING DOWN AND
SPEEDING UP REALLY HELPS, SO
THINKING ABOUT THESE AS MORE
TEMPLATES DISSEMINATED TO THE
COMMUNITY MIGHT ALSO BE HELPFUL.
BUT WE'RE REALLY LEARNING FROM
THE COMMUNITY ON HOW TO BUILD
THEM WELL.
AND I THINK THAT'S THE IMPORTANT
PIECE HERE FROM US.
AND I THINK AS WE'VE DONE THAT
WE HAVE, GOING BACK TO SERGIO'S
COMMENT ON TRUST, AND
TRUSTWORTHINESS, YOU KNOW,
BECAUSE WE'RE LISTENING TO THE
COMMUNITY AND THEY ARE SEEING
THAT BUILT INTO THE AGREEMENTS,
THAT ULTIMATELY HELPS US SO THAT
WE HAVE BECOME A MORE FAMILIAR
PARTNER AND COLLABORATIVE TO
WORK WITH.
THERE'S STILL MORE TO GO, IN
TERMS OF DISSEMINATION, AND
SOMETHING THAT WE CERTAINLY
THINK ABOUT QUITE A BIT ON HOW
WE MIGHT BE ABLE TO DO THAT.
ANIKA?
>> GREAT PRESENTATION.
I ECHO WHAT'S BEEN SAID.
IT'S JUST GREAT SEEING ALL THE
PRESENTATIONS, HOW THEY HAVE
BEEN BUILDING, YOU KNOW, ONTO
THINGS THAT WERE DISCUSSED
EARLIER IN THE YEAR AND COMING
TO FRUITION.
SAME WITH DAN'S, REALLY NICE TO
SEE.
I WAS INTRIGUED BY YOUR LAST
SLIDE, YOU KNOW, JUST -- HOW
YOU'RE USING THE I-CORPS MODEL
TO TRAIN ENTREPRENEURS AND SMALL
BUSINESSES IN HOW TO, YOU KNOW,
MOVE THEIR EFFORTS FORWARD.
AND IT REALLY -- YOU KNOW, YOU
OFTEN HEAR ABOUT, YOU KNOW, ONE
OF THE TRANSLATIONAL SCIENCE
ISSUES IS YOU HAVE PEOPLE WHO
ARE GREAT SCIENTISTS BUT THEY
ARE NOT GREAT BUSINESS PEOPLE.
AND SO IT KIND OF GETS TO THAT
POINT OF HOW DO YOU DO TRAINING
OF THESE SCIENTISTS AND
ENGINEERS SO THEY KNOW, YOU
KNOW, HOW TO MOVE FORWARD THEIR
BUSINESS.
AND, YOU KNOW, YOU SEEM LIKE YOU
GAVE CASE STUDIES OF THAT.
I GUESS I'M WONDERING IF -- LIKE
IT MADE ME THINK ABOUT, YOU
KNOW, BUSINESSES THAT CAME OUT
OF LIKE THE TISSUE CHIPS
PROJECT.
YOU KNOW, ARE YOU KIND OF
BUILDING THEIR KNOWLEDGE AND
UNDERSTANDING HOW TO MOVE
FORWARD AND HANDLE I.P. AND THEN
PAULA TRIGGERED THE SAME IDEA
TRAINING ENTREPRENEURS AS WE'RE
DEVELOPING THESE NEW AGREEMENTS,
AND THESE NEW DATA PLATFORMS
THAT COULD ACCELERATE THEIR
BUSINESSES, YOU KNOW, IS THAT
PART OF MAYBE YOUR SHOP IN TERMS
OF HOW TO DO THE TRAINING?
SO I GUESS IN SUMMARY, THERE WAS
A LOT OF PRESENTATION ON HOW
YOUR OFFICE HAS BEEN USED TO
ACCELERATE NCATS WORK AND BECOME
PREFERRED PARTNER BUT I GUESS
THE OTHER PIECE OF THAT IS HOW
DO YOU KIND OF BUILD THE
CAPACITY OF THE SMALL BUSINESSES
AND ENTREPRENEURS OUT THERE SO
THEY CAN TRAVERSE THE VALLEY OF
DEATH EVEN BETTER.
PARTICULARLY AS THESE NEW TOOLS
AND PLATFORMS AND YOUR SHOP
BUILDING ALL THESE AGREEMENTS
MAKING IT EASIER TO DO
TRANSLATIONAL SCIENCE AND DRUG
DEVELOPMENT JUST KIND OF
BUILDING CAPACITY IN THAT
REGARD.
THE FLIP SIDE, YOU KNOW, THOSE
ARE SMALL BUSINESSES, ALSO
THINKING THE FLIP SIDE OF THE
VENTURE CAPITALISTS, THEIR ROLE
BUILDING THEIR KNOWLEDGE, HOW TO
INTERFACE WITH SMALL BUSINESSES
IN BIOMEDICAL RESEARCH, AND THEN
WHICH MIGHT BE HARDER, I THINK
EVEN WITH LARGE COMPANIES
THERE'S INTERNAL PROCESSES THAT
ARE BOTTLENECKS WHEN IT COMES TO
BUSINESS AND LEGALITY AND SO IS
THERE SOMETHING THERE SO
BASICALLY USING LEVERAGING YOUR
EXPERTISE, YOU KNOW, THE
BUSINESS AND LEGAL SIDE IS IN
AND OF ITSELF THAT NEEDS TO BE
IMPROVED TO HELP ADVANCE
TRANSLATIONAL SCIENCE, SO,
AGAIN, THAT COULD BE THE REALM
OF BUILDING CAPACITY OF THE
ENTREPRENEURS AND SMALL
BUSINESSES AS YOU'RE DOING AND
REALLY LOOKING AT THOSE PIECES
AS WELL AS HOW TO GET OVER THAT
VALLEY OF DEATH.
>> YEAH, THANKS, ANIKA.
I'LL ANSWER IN TWO PARTS.
ONE IS IN TERMS OF SOME OF THE
THINGS WE DO IN OUR OFFICE, IF
WE FIND SOMETHING INTERESTING OR
IF WE FIND SOMETHING UNIQUE WE
TRY TO PUBLISH IT AND PRESENT
DIFFERENT TRADE CONFERENCES.
FOR INSTANCE CRCA WE PUBLISHED
AND PRESENTED.
SIMILARLY OTHER SITUATIONS WHERE
WE'VE DONE INTERESTING
COLLABORATIONS, LICENSED,
PRESENTED AT TRADE CONFERENCES
LIKE THE ASSOCIATION OF
UNIVERSITY TECHNOLOGY MANAGERS.
IN TERMS OF SMALL BUSINESS
COMMUNITY THINKING AND TRAINING
THEM ALREADY A LOT OF SUPPORTIVE
PROGRAMS THAT NIH RUNS.
FOR INSTANCE, THE MAIN NIH SMALL
BUSINESS EDUCATION OFFICE, RUN
BY OFFICE OF THE DIRECTOR, THEY
HAVE PROGRAMS WHERE
ENTREPRENEURS WHO HAVE NEVER
APPLIED OR RECEIVED SBIR
PROGRAM, THEY CAN GO THROUGH
TRAINING PROGRAM CALLED AAP,
APPLICANT ASSISTANCE PROGRAM.
DURING THE PROJECT ITSELF, THEY
CAN GET CONSULTATIONS FROM
VARIOUS GROUPS, IF SOMETHING
NEEDS TO BE PATTENEDDED THEY
CAN -- PATENTED THEY CAN GET
POINTERS, EVEN AFTER THEY CAN GO
THROUGH I-CORPS TRAINING TO SEE
IF COMMERCIALIZATION THINKING IS
ALONG THE RIGHT WAY OR NOT.
AND SOME COMPANIES HAVE PIVOTED
AND WHEN THEY COME BACK TO NCATS
WITH A PHASE 2 APPLICATION THEIR
APPLICATION IS MUCH, MUCH
STRONGER BECAUSE THEY HAVE GONE
THROUGH THE I-CORPS PROGRAM AND
THOUGHT ABOUT HOW TO MOVE
FORWARD.
WE ALSO HAVE INTERNAL
EDUCATIONAL SERIES WHERE ONCE IN
THREE OR FOUR MONTHS OUR OFFICE
WILL RUN SEMINARS ABOUT
PATENTING, LICENSING, DIFFERENT
FINANCIAL MODELS, ABOUT COMPANY
FORMATION, ET CETERA.
BUT THESE ARE MOSTLY INTERNAL
SEMINARS FOR NCATS STAFF.
WE ARE A FAIRLY SMALL OFFICE,
HAVE TO FOCUS ON WHAT WE DO.
>> YEAH, YEAH.
MAYBE TO YOUR POINT, LIKE IF NIH
IS ALREADY DOING TRAININGS FOR
ENTREPRENEURS, THROUGH SMALL
BUSINESS PROGRAMS, MAKING YOUR
INNOVATIVE INSTRUMENTS THAT YOU
CREATED HAVE BEEN INCLUDED IN
THOSE TRAININGS, YOU KNOW, LIKE
THE CRCA, AND OTHER THINGS, SO
THAT THESE YOUNG SCIENTISTS AND
BUSINESSES KNOW ABOUT THESE NEW
TOOLS OF HOW THEY CAN CREATE
AGREEMENTS AND DO BUSINESS,
BECAUSE YOU GUYS HAVE DONE SOME
GREAT WORK.
SO MAYBE THAT'S A WAY TO KIND OF
DEAL WITH SMALL CAPACITY STAFF.
>> THANK YOU.
WE TRY TO LEVERAGE AS MUCH AS WE
CAN ON THE OUTSIDE WHAT WE CAN.
>> MARSHALL?
>> I'LL MAKE THIS QUICK.
WHAT YOU'RE DOING IS REALLY
GOOD.
I'VE HAD INTERACTIONS ON SOME
DEVICES.
THE PROBLEM I RUN INTO TIME AND
TIME AGAIN THE TECHNOLOGY
TRANSFER OFFICES AT MOST
UNIVERSITIES ARE OFTEN STAFFED
BY VERY JUNIOR FOLK, THE LEGAL
FOLKS THEY ASSIGN TO THEM
TYPICALLY HAVE THE HALF-LIFE OF
A MAY FLY, I.E. A DAY OR TWO.
AND THEY DON'T REALLY HAVE MUCH
INSTITUTIONAL KNOWLEDGE, NOR DO
THEY KNOW WHERE TO GET IT FROM.
AND THAT ACTUALLY I FIND
PARTICULARLY WHEN WORKING WITH
NIH TRYING TO GET NIH-FUNDED
RESEARCH OUT THERE INTO THAT, IF
THERE'S A WAY TO INCREASE WITH
YOUR ALREADY SMALL OFFICE THE
AMOUNT OF SORT OF TEACHING
MATERIAL FOR THESE TECHNOLOGY
TRANSFER GROUPS OUT THERE IN THE
REAL WORLD, THEY NEED -- I MEAN,
THEY WILL CLAIM TO HAVE
PRESERVED A LIFE PRESERVER, SOME
PLACES DO IT WELL BUT THE
MAJORITY I'VE WORKED WITH OVER
THE YEARS, I WAS COUNTING THE
OTHER DAY 130 PATENTS THEY HAVE
DONE DIFFERENT THINGS WITH, I
HAVEN'T SEEN A ONE OF THEM GO
SMOOTHLY BASED ON THE
UNIVERSITY'S TECHNOLOGY
TRANSFER.
>> I HEAR YOU, MARSHALL.
ONE OF THE CHALLENGES FOR ANY
TECHNOLOGY TRANSFER OFFICE IN A
UNIVERSITY FOR INSTANCE IS THEY
TEND TO BE SEPARATE, ISOLATED
OFFICE.
>> YEAH, THEY ARE.
>> THEY ARE, RIGHT?
AND THEY ARE DEALING WITH
ENGINEERING, SCIENCE, ALL KINDS
OF DISCIPLINES.
AT NCATS, AND NIH, SPECIFICALLY
NCATS, WE'RE VERY LUCKY THAT
WE'RE EMBEDDED WITH SCIENTISTS,
VERY MISSION FOCUSED, MISSION
ORIENTED.
PEOPLE IN MY OFFICE HAVE BEEN
WITH US FOR A LONG TIME.
>> SHARE YOUR BEST PRACTICES
WITH THE COMMUNITY.
I KNOW THAT'S ONE MORE JOB.
YOU DON'T NEED ANOTHER JOB, I
GET IT.
>> I'LL BE HAPPY TO SHARE MANY
SECRETS.
ONE OF THE BIGGEST SECRET SAUCE
IF YOU'RE IN AN ORGANIZATION
LIKE NCATS, VERY TEAM ORIENTED,
IT MAKES THE LIFE OF TECH
TRANSFER OFFICE MUCH EASIER.
>> IT DOES.
>> BECAUSE YOU'RE SO SURROUNDED
BY PEOPLE YOU CAN GO TO, BECAUSE
MANY TECH TRANSFER OFFICES WHEN
THEY TRY TO SOLVE PROBLEMS THEY
ARE ON THEIR OWN.
>> YEAH.
>> A SITUATION I CANNOT SOLVE IT
MYSELF, I CAN GO DOWN THE ROAD
AND TALK TO ANTON, SAM, AND SAY
THIS IS SOMETHING I NEED HELP
WITH.
>> I DON'T WANT YOU TO RUN A
SUPPORT GROUP FOR THEM ALTHOUGH
THAT WOULD BE GREAT.
IF NOTHING ELSE, IF THERE'S A
WAY TO GET SOME LESSONS LEARNED
AND BEST PRACTICES OUT THERE IN
SOME USEFUL FORMAT THIS WOULD BE
POWERFUL.
>> TO THAT SUGGESTION, MARSHALL,
TECH TRANSFER 101 MIGHT BE
HELPFUL, SOMETHING TO THINK
ABOUT FOR SURE.
THANK YOU.
AS ALWAYS, THESE ARE GREAT
COMMENTS.
BUILDING THAT COMMUNITY, IN
ADDITION TO SHARING THAT
KNOWLEDGE, IS CERTAINLY
SOMETHING THAT WE WOULD WANT TO
DO.
>> IF THESE THINGS DON'T MAKE A
TRANSITION TO COMMERCIAL
APPLICATION, MOST PATIENTS WILL
NEVER SEE THEM.
>> YEAH, YEAH.
THAT'S ULTIMATELY WHAT WE WANT,
RIGHT.
>> ONE OF THE THINGS, MARSHALL,
I LEARNED ALONG THE WAY IS THAT
SOME THINGS, WHEN YOU TEACH HOW
TO BE A BETTER NEGOTIATOR YOU
CAN GET A GOOD AGREEMENT
TOGETHER.
>> OH, YEAH.
LET ME ALSO KNOW WHAT YOU CAN
AND CAN'T DO, ALL SORTS OF
THINGS LIKE THAT.
>> EXACTLY.
KELLY?
>> OH, I PUT MY HAND DOWN IN THE
INTEREST OF TIME.
I WAS GOING TO ASK YOU IF YOU --
FIRST I WANT TO SAY HOW
VISIONARY AND HEROIC TO HAVE
INNOVATED FOR COLLABORATION
AGREEMENTS.
I FELT THAT WAS SO INTERESTING.
I WONDERED IF YOU HAVE BEEN
EXPERIMENTING OR EXPLORING THE
POSSIBILITY OF USING SMART
CONTRACTS TO AUTOMATE SOME
ROYALTY AGREEMENTS AND OTHER
KINDS OF AGREEMENTS.
I'LL PAUSE THERE AND ASK THAT
QUESTION.
>> CAN YOU ELABORATE?
DO YOU MEAN USING THIRD PARTIES?
>> NO, ACTUALLY -- THAT'S WHY I
DIDN'T -- THAT'S WHY I LOWERED
MY HAND.
WHAT I'M GOING TO DO RATHER
THAN -- I'M GOING TO CEDE THE
FLOOR AND SEND AN E-MAIL.
IT'S TOO LONG.
>> THAT WORKS.
THAT WOULD BE GREAT.
THANK YOU FOR THE FOLLOW-UP AND
CONNECTION.
SOUNDS LIKE IT WOULD BE VERY
INTERESTING, FROM THAT LITTLE
BIT I HEARD.
AND THEN A COUPLE COMMENTS HERE
IN THE CHAT, BALKI.
PAUL HARRIS MENTIONED YOU MIGHT
CONSIDER A PUBLICATION, SHARING
LESSONS LEARNED.
MARSHALL LIKED THAT IDEA AS
WELL, SINCE IT'S A STUMBLING
BLOCK.
YEAH, THANK YOU.
AND ANIKA IS PULLING UP SOME
TECH TRANSFER EDUCATION
RESOURCES WE HAVE ON OUR WEBSITE
TOO.
SO MAYBE A COMBINATION OF BOTH
OF THOSE THINGS, THINKING ABOUT
HOW TO DISSEMINATE THAT TO THE
COMMUNITY WOULD BE HELPFUL.
ALWAYS SOMETHING TO THINK ABOUT
AND CHEW ON HERE.
ANY OTHER COMMENTS FOR BALKI?
AND IF NOT, THANK YOU SO MUCH
FOR THAT OVERVIEW, BALKI.
WE HAD A TOUR DE FORCE TODAY.
IT SEEMED THE THEME HAS BEEN
REALLY FOCUSED AROUND TRUST AND
TRUSTWORTHINESS, AND HOW TO
INCLUDE THAT FUNDAMENTALLY IN
WHAT WE DO.
THE FACT THAT IT HAS COME UP A
LOT IS A GOOD SIGN.
AND BUT CLEARLY WE HAVE MORE TO
DO.
WE TALKED ABOUT HOW IT CAN BE
INFUSED IN DATA SCIENCE,
PRE-CLINICAL MODELS, TECH
TRANSFER.
EVERY ASPECT WE TOUCH WILL BE
IMPORTANT.
ALSO TALKING ABOUT THE -- HOW TO
BRING THE NAMs CAREFULLY MORE
INTO THE CLINIC, AND ENGAGING
PATIENTS IN THAT WORK TOO.
ALSO AN IMPORTANT THING TO THINK
ABOUT AND CONTRIBUTION, THINKING
ABOUT A.I. AND M.L. ENABLED
WORK, CAPTURING INSIGHTS INTO
THE LITERATURE WE CAN'T
NECESSARILY SEE OURSELVES,
SOUNDS LIKE THERE'S GREAT GROUND
BEING MADE THERE.
IT WAS GREAT TO HAVE MONICA
BERTAGNOLLI GIVE HER VISION AND
OVERSIGHT OF NIH NOW AT THE
HELM, TALKING ABOUT HER GUIDING
PRINCIPLES, AND DESPITE THE FACT
WE HAVE SOME BUDGET CONCERNS,
YOU KNOW, WE'RE GOING TO DO THE
BEST WITH WHAT WE HAVE, HOW TO
PRIORITIZE AND RESTORE SOME
TRUST AGAIN CAME UP IN THAT
CONVERSATION.
I'M EXCITED TO SEE HOW THAT
UNFOLDS.
WITH DAN AND BALKI'S
PRESENTATION, IT'S GOOD TO HAVE
THE UPDATES, I APPRECIATE YOUR
COMMENTS AND FEEDBACK ON THAT.
AND I DON'T HAVE ANYTHING MORE
TO SAY EXCEPT I REALLY
APPRECIATE AS ALWAYS THE
IMPORTANT CONTRIBUTIONS THAT OUR
COUNCIL MAKES AND JUST SAY ONCE
MORE, AGAIN, THANK YOU TO
MATTIAS AND MARSHALL FOR YOUR
LASTING IMPRINT ON NCATS, AND I
LOOK FORWARD TO SEEING THE REST
OF OUR COUNCIL MEMBERS AT OUR
NEXT MEETING IN MAY.
WITH THAT, I WILL GAVEL US OUT
OF OUR MEETING TODAY.
THANK YOU SO MUCH AGAIN. 5578 03:37:04,411 [END OF PROGRAM]