>>I WANT TO WELCOME EOPLE TO
THE 218TH MEETING OF THE
NATIONAL ADVISORY COUNCIL FOR
THE NEUROLOGICAL DISORDERS AND
STROKE.
I WANT TO MENTION I THINK WE'RE
FIVE AWAY FROM 2025, WHICH IS
OUR 75TH ANNIVERSARY.
WE'RE ALREADY THINKING OF TRYING
TO DO SOMETHING SPECIAL FOR THAT
MOMENTOUS OCCASION TO KIND OF
CELEBRATE AND THINK FORWARD
ABOUT THE GREAT STUFF OUR
GRANTEES AND STAFF HAVE DONE
OVER THE LAST 75 YEARS AND ARE
POISED TO DO IN THE FUTURE.
FIRST THING I WANT TO DO IS TO
INTRODUCE THE COUNCIL AD HOCS.
THESE ARE FOLKS THAT HAVE THEIR
APPLICATION DOWNTOWN TO HH S AND
HOPEFULLY WILL BE COMING ON
OFFICIALLY VERY SHORTLY.
I CAN JUST SEE THE DESKS
DOWNTOWN WITH ALL THESE STACKS
OF PAPER AND SOMEWHERE IN THERE
IS HANK OR HENRY GREELEY, JOHN
SORE PROFESSOR OF LAW AT
STANFORD UNIVERSITY, ALSO
PROFESSOR BY COURTESY IN
GENETICS AT STANFORD SCHOOL OF
MEDICINE.
HE SPECIALIZES IN ETHICAL, LEGAL
AND SOCIAL IMPLICATION OF NEW
BIOMEDICAL TECHNOLOGIES,
PARTICULARLY THOSE RELATED TO
GENETICS ASSOCIATED WITH
REPRODUCTION, NEUROSCIENCE, AND
STEM CELL RESEARCH.
HANK WAS A FOUNDING MEMBER OF
THE NIH BRAIN MULTI-COUNCIL
WORKING GROUP AND THE FOUNDING
CHAIR OF THE BRAIN NEUROETHICS
WORKING GROUP, AND HE'S ALSO A
FOUNDER AND PAST PRESIDENT OF
THE INTERNATIONAL NEUROETHICS
SOCIETY, HE RECEIVED HIS LAW
DEGREE FROM YALE LAW SCHOOL AND
HE HAS A SLIGHT DEFORMITY OF HIS
LIP, HE'LL BE TWISTING IT ALL
THE TIME HAVING TO ASK HIM TO DO
THINGS FOR NANDS, SO REALLY
LOOKING FORWARD TO HAVING HANK
JOINING US.
NEXT IS HENRY PAULSON, WHO ALSO
GOES BY HANK.
PROFESSOR OF NEUROLOGY FOR
ALZHEIMER'S DISEASE AND RELATED
DEMENTIAS, IN THE DEPARTMENT OF
NEUROLOGY AT THE UNIVERSITY OF
MICHIGAN.
HE DIRECTS THE MICHIGAN
ALZHEIMER'S DISEASE CENTER AND
HE CO-DIRECTS THE MICHIGAN
NEUROSCIENCE INSTITUTE.
HANK ALSO WAS THE CHAIR OF THE
NINDS INTRAMURAL BOARD OF
SCIENTIFIC COUNSELORS, SO HE
KNOWS OUR INTRAMURAL PROGRAM
WELL, AND HE RECEIVED HIS
MEDICAL AND DOCTORATE DEGREE
ALSO FROM YALE.
NEXT IS JANE LARKINDALE, VICE
PRESIDENT OF CLINICAL SCIENCE AT
P E-PG EN, A BIOTECH COMPANY
FOCUSING ON DEVELOPING
TREATMENTS FOR NEUROMUSCULAR
DISEASES.
PRIOR TO JOINING PEP GEN, SHE
WORKED FOR THE EXECUTIVE
DIRECTOR FOR THE RARE DISEASE
CURES ACCELERATOR DATA AND
ANALYTICS PLATFORM AT THE
CRITICAL PATH INSTITUTE, WHICH
WE'LL BE TALKING ABOUT LATER
THIS AFTERNOON.
SHE IS A MOLECULAR BIOLOGIST BY
TRAINING, COMPLETING HER PH.D.
AT OXFORD.
AND SHE ATTENDED ON A RHODES
SCHOLAR SHIP.
NEXT, PLEASED TO INTRODUCE
JIN-MOO LEE, PROFESSOR OF
NEUROLOGY AT WASHINGTON
UNIVERSITY SCHOOL OF MEDICINE IN
ST. LOUIS AND ALSO PROFESSOR OF
RADIOLOGY, AND HE'S THE CHAIR OF
THE DEPARTMENT OF NEUROLOGY AND
NEUROLOGIST IN CHIEF AT BARNES
U.S. HOSPITAL.
PRIOR TO BECOMING THE DEPARTMENT
CHAIR, DR. LEE WAS THE CO-CHAIR
FOR THE STROKE CARE CLINICAL
PROGRAM AT BJC HEALTHCARE IN
MISSOURI.
HIS LAB FOCUSES ON
PATHOPHYSIOLOGY AND CELLULAR
MOLECULAR MECHANISMS INVOLVED IN
BRAIN INJURY FOLLOWING ACUTE
ISCHEMIA AND CHRONIC
NEURODEGENERATION.
HE EARNED HIS M.D. AND PH.D. IN
NEUROSCIENCE AT WEILL CORNELL
MEDICAL COLLEGE, LOOKING FORWARD
TO HIM JOINING.
LAST TO ANNOUNCE IS YISHI JIN,
GOES BY JIN, PROFESSOR IN THE
DEPARTMENT OF NEUROBIOLOGY AND
DEPARTMENT OF CELLULAR AND
MOLECULAR MEDICINE.
SHE HOSHE'S CO-DIRECTOR OF THE Y
INSTITUTE OF BRAIN AND MIND BASE
AT UCSD.
HER RESEARCH FOCUSES ON
NEURODEVELOPMENT IN THE C.
ELEGANS, AND SHE RECEIVED THE
NSF PRESIDENTIAL EARLY CAREER
AWARD FOR SCIENTISTS AND
ENGINEERS, AND SHE COMPLETED HER
PH.D. IN MOLECULAR BIOLOGY AT
THE UNIVERSITY OF CALIFORNIA,
BERKELEY.
SO WHAT A GREAT GROUP WE HAVE
JOINING, AND THANKS IN ADVANCE
FOR ALL THE WORK WE'LL GIVE YOU
TO DO!
SO WITH THAT, BOB, I'LL TURN IT
OVER TO YOU.
>>THANKS A LOT, WALTER.
AND IT IS REALLY GREAT TO SEE
EVERYBODY.
NEXT TIME HOPEFULLY IN PERSON.
THAT WILL BE GREAT.
JUST MENTIONING ONE THING ABOUT
THE NEW COUNCIL AD HOCS, AD HOC
MEANS YOU CAN PARTICIPATE FULLY
IN ALL THE DISCUSSIONS, YOU JUST
CAN'T FORMALLY VOTE WHEN WE VOTE
ON THINGS, AND THAT'S ALSO TRUE
IN THE CLOSED SESSION TOMORROW.
BUT IT'S GREAT TO HAVE ALL OF
YOU.
JUST MY USUAL REMINDER THAT THIS
IS ALL BEING VIDEOCAST AND
ARCHIVED, SO LOOK GOOD, BE GOOD,
ET CETERA, ET CETERA.
EVERYBODY HAS ZOOM DOWN BY NOW
SO I'M NOT GOING TO SAY MUCH
ABOUT THAT.
I'LL JUST SAY THAT IF YOU WANT
TO SAY ANYTHING OR MAKE A
MOTION, YOU CAN EITHER JUST
SPEAK UP OR USE YOUR EMOJI HAND.
IT'S BETTER WHEN YOU USE YOUR
EMOJI HAND THAN YOUR REAL HAND
BECAUSE IT PUTS YOU IN A PLACE
WHERE I CAN SEE YOU.
YOU'LL BE VOTING IN THE CHAT.
TIM IS GOING TO ENTER EVERY
MOTION IN THE CHAT AND THEN YOU
CAN VOTE.
ACCORDINGLY.
OKAY.
SO THE FIRST THING I NEED IS
YOUR APPROVAL OF THE MINUTES
FROM THE LAST COUNCIL MEETING,
WHICH ARE IN THE ECB.
AND UNLESS ANYONE WANTS TO
DISCUSS THE MINUTES?
I'LL PAUSE FOR A SECOND.
I NEED A MOTION TO APPROVE THEM.
>>SO MOVED.
>>I SEE TIM.
IS THERE A SECOND?
>>SECOND.
>>GOING ONCE, GOING TWICE,
OKAY, EVERYONE VOTE IN THE CHAT.
LOUISE, DO YOU HAVE A QUESTION?
OH, YOU WERE RAISING YOUR EMOJI
HAND FOR -- OKAY.
OKAY.
SO WE SUCCESSFULLY APPROVED THE
MINUTES.
THAT'S ALWAYS GREAT TO GET OFF
TO A GOOD START.
NEXT THING IS THE DATES FOR
FUTURE COUNCIL MEETINGS.
I ALREADY MENTIONED THE DATES
FOR THE NEXT ONE.
NOTICE THAT WE'RE GOING TO
CONTINUE TO HAVE THEM ON
WEDNESDAYS AND THURSDAYS.
ON TWO CONSECUTIVE DAYS.
SO IF YOU HAVE A PROBLEM WITH
ANY OF THESE DATES, DO LET TIM
KNOW BECAUSE WE NEED TO PLAN
ACCORDINGLY.
IN TERMS OF MAY, WE REALLY DO
WANT TO HAVE THE MEETING IN
PERSON.
OBVIOUSLY THIS DEPENDS ON COVID
TRENDS, BUT WE'LL KEEP IN TOUCH
AND LET YOU KNOW.
AND WE'RE ACTUALLY THINKING THAT
WE SHOULD HAVE AT LEAST ONE
MEETING IN PERSON A YEAR, BUT
WE'LL DISCUSS THE DETAILS OF
THAT MORE LATER.
OKAY.
I'M GOING TO GO ON TO ALL MY
BOILERPLATE STUFF.
THE FIRST THING, AND I'LL TALK
FAST BECAUSE THERE'S ACTUALLY A
LOT THAT I NEED TO SAY.
SO THE FIRST THING IS THE
EXPEDITED REVIEW PROCESS, SO AS
YOU KNOW, WE REPORT ON THIS
PROCESS OR ARE REQUIRED TO
REPORT ON THIS AT EVERY COUNCIL
MEETING.
THIS IS WHEN A SUBSET OF
COUNCILMEMBERS APPROVE
APPLICATIONS IN ADVANCE OF THE
NORMAL SCHEDULE, IN FACT IN
ADVANCE OF THE COUNCIL MEETING
FOR FUNDING, AND THAT ALLOWS OUR
GRANTS MANAGEMENT BRANCH TO
ISSUE THEM MORE QUICKLY SO THE
RESEARCH CAN GET STARTED.
WE USUALLY FOCUS ON THINGS LIKE
R01s WITHIN THE PAYLINE, WHICH
HAVE NO ANIMAL ISSUES OR HUMAN
SUBJECTS ISSUES.
SO THEY GOT GREAT SCORES AND WE
JUST WANT TO GET THEM FUNDED.
WE ROTATE THE TASK AMONG THREE
COUNCILMEMBERS EVERY TIME.
THIS TIME AROUND, IT WAS -- THIS
YEAR IT'S KEN, JOHN AND H EKEMI.
WE'RE WELL ON TRACK TO ISSUING
THESE OR ISSUING THEM VERY SOON.
EVERY TIME WE CAN DO THIS AHEAD
OF SCHEDULE BECAUSE OF OUR GREAT
GRANTS MANAGEMENT BRANCH LED BY
SHELLEY WHO YOU'LL HEAR FROM IN
A MOMENT AND ALSO TO OUR PROGRAM
STAFF WHO DO A GREAT JOB MAKING
SURE THESE APPLICATIONS MEET THE
CRITERIA FOR BEING EXPEDITED.
OKAY.
SO IN TERMS OF ANNOUNCEMENTS, WE
USED TO INTRODUCE ALL OUR NEW
STAFF BUT IN THESE STREAMLINED
SHORTENED VIRTUAL MEETINGS, WE
JUST PUT THEIR DESCRIPTIONS IN
THE ECB.
I THINK THERE'S ACTUALLY A SLIDE
LISTING ALL OF THE NEW STAFF, IS
THAT RIGHT?
THERE GOES KEVIN.
SO YOU CAN SEE, THERE'S A COUPLE
IN THE DIVISION OF CLINICAL
RESEARCH, A FEW IN TRANSLATIONAL
RESEARCH, A BUNCH IN MY
DIVISION, WHICH IS EXTRAMURAL
ACTIVITIES, AND ALSO IN OUR
COMMUNICATIONS OFFICE AND
FINALLY THE BIGGEST GROUP IS IN
OUR DIVISION OF NEUROSCIENCE.
SO ALL THESE PEOPLE HAVE VERY
IMPRESSIVE BIOS, AND YOU'RE
WELCOME TO READ ABOUT THEM IN
THE ECB.
OKAY.
KEVIN, YOU CAN TAKE THAT DOWN.
SO THE ONE THING I DON'T WANT TO
SKIP IS SAYING GOODBYE TO SOME
REALLY GREAT PEOPLE WHO ARE
LEAVING.
WE'LL DO THAT RELATIVELY QUICKLY
BUT I THINK IT'S IMPORTANT TO
MENTION THESE PEOPLE WHO HAVE
MANY YEARS OF DEDICATED SERVICE
TO OUR INSTITUTE.
THE FIRST ONE, I HOPE HE'S IN
THE MEETING.
THE FIRST ONE IS GOING TO BE
SPOKEN OF BY ERNIE LYONS, THE
HEAD OF OUR REVIEW BRANCH, ONE
OF OUR TERRIFIC SROs IS SADLY
LEAVING, ACTUALLY IT'S FOR A
PROMOTION BUT I'LL LETTER KNEE
EXPLALET ERNIEEXPLAIN ALL OF TH.
HI, ERNIE.
>>HELLO, EVERYONE.
AND MARILYN IS ON AS WELL, I
BELIEVE.
>>I SEE HER.
>>OKAY, GREAT.
SO I'M THE CHIEF OF REVIEW HERE
AT NINDS.
I'VE BEEN HERE FOR A LONG TIME.
I KNOW MANY OF YOU.
SO I WANTED TO TAKE A MINUTE TO
JUST ACKNOWLEDGE AND THANK
MARILYN MOOREHOON, SRO IN OUR
BRANCH FOR THE PAST 4 1/2 YEARS.
SHE JOINED US AS AN EXPERIENCED
SRO.
SHE HAD BEEN A SRO PREVIOUSLY
FOR ABOUT EIGHT YEARS AND I'D
KNOWN MARILYN FOR A LONG TIME
THROUGH OUR WORK ON TRANS-NIH
COMMITTEE, I WAS ALWAYS VERY
IMPRESSED WITH HER WORK.
MARILYN JOINED US DURING THIS
TIME OF RAPID EXPANSION IN SRB.
WE WERE TAKING ON REVIEW OF ALL
THESE NEW PROGRAMS, THE BRAIN
INITIATIVE, ADRD, THE HEAL
INITIATIVE.
AND WE EXPANDED OUR BRANCH,
FORMED NEW GROUPS MUCH LIKE SCR
REVIEW TEAMS AND MARILYN WAS ONE
OF OUR INITIAL TEAM LEADS.
SHE'S AN EXTREMELY TALL EBT
ENTED SRO, SHE RAN MANY OF OUR
VERY COMPLICATED PROGRAMS,
CENTERS OF EXCELLENCE FOR
PARKINSON'S DISEASE AND MANY
OTHERS.
SHE'S EXTREMELY KNOWLEDGEABLE
ABOUT REVIEW POLICY AND
PROCEDURES AND PROCESS, AND SHE
WAS TREMENDOUSLY HELPFUL TO THE
SRB DURING THE EXPANSION, AND
HAS REALLY DONE A LOT TO SUPPORT
THE MISSION AT NINDS IN THESE
PAST 4 1/2 YEARS.
AND SHE HAS JUST LEFT US IN
JANUARY, STARTING THIS YEAR.
SHE'S NOW AT THE NATIONAL CENTER
FOR ADVANCING TRANSLATIONAL
SCIENCE, WHERE SHE IS NEW REVIEW
CHIEF AND I'M SURE SHE'LL
CONTINUE TO DO OUTSTANDING WORK
AS THE CHIEF OF SRB THERE, SO
CONGRATULATIONS, MARILYN, AND
THANK YOU FOR ALL OF YOUR
OUTSTANDING WORK.
>>YES.
>>THANK YOU.
>>WE WILL REALLY MISS YOU,
MARILYN.
>>AND I'LL MISS YOU GUYS TOO.
>>YES.
I'M ALWAYS A LITTLE NERVOUS
AROUND MARILYN BECAUSE SHE'S SO
SHARP.
YOU REALLY HAVE TO BE ON YOUR
TOES WHEN YOU'RE TALKING TO HER.
OKAY.
THAT'S MARILYN.
NEXT, SHELBY WILBURN IS GOING TO
SPEAK BRIEFLY ABOUT TWO PEOPLE
THAT ARE SPEAKING.
IS SHELLEY ON SOMEWHERE?
OKAY, TIM, MAKE SURE SHELLEY HAS
AN INVITE TO THE MEETING.
I'LL GO ON TO THE NEXT ONE WHILE
WE'RE TRYING TO FIGURE THAT OUT.
>>I THINK SHELLEY IS GOOD TO GO
NOW.
>>OKAY.
IS SHE ON THE SCREEN?
I'M NOT SEEING HER.
BUT CAN WE HEAR SHELLIE?
CAN YOU SAY SOMETHING?
>>I JUST GOT ACCESS.
SORRY ABOUT THAT.
SO YES.
TWO DEPARTURES SINCE THE LAST
COUNCIL MEETING.
THE FIRST IS OLAMIDA ADENEA WHO
MANAGED OUR BRAIN PORTFOLIO.
SHE WAS DEFINITELY A TRUE ASSET
TO THE ORGANIZATION.
SHE HAD BEEN HERE FOR 14 YEARS.
AND SHE WILL DEFINITELY BE
MISSED WITHIN THE ORGANIZATION.
AND THE SECOND IS ERNA
PETHRIDGE, HELPING US WITH OUR
TRANSACTIONS AWARDS WITHIN THE
ORGANIZATION.
SHE WAS ONLY HERE A FEW YEARS
BUT SHE MADE A TREMENDOUS IMPACT
ON THE ORGANIZATION AS WELL, AND
THEY WILL BOTH BE MISSED AND
THEY'RE BOTH IRREPLACEABLE.
THANK YOU.
>>BOTH ABSOLUTELY TERRIFIC
EMPLOYEES.
LAST BUT NOT LEAST IS CLINT
WRIGHT, THE HEAD OF OUR CLINICAL
RESEARCH DIVISION, IS GOING TO
TALK ABOUT ANOTHER SAD,
BITTERSWEET DEPARTURE.
>>BITTERSWEET INDEED.
I WANTED TO THANK JANICE CORDELL
ON HER RETIREMENT.
SHE RETIRED AFTER 34 YEARS OF
SERVICE AT NIH.
SHE HAD BEEN WITH THE DIVISION
OF CLINICAL RESEARCH SINCE 2002,
AS A NURSE CONSULTANT AND
CLINICAL RESEARCH PROJECT
MANAGER, AND SHE SAW A LOT OF
PROGRAMS FROM THE START,
INCLUDING NEURONEXT, OUR GENERAL
NEUROLOGY CLINICAL TRIAL NETWORK
BUT SHE ALSO LED DSMBs AND WAS
VERY, VERY HELPFUL THROUGH MANY
DISCUSSIONS WITH OTHER GROUPS
INCLUDING INDUSTRY AND OTHER
PARTNERSHIPS WITH PRIVATE
FOUNDATIONS.
SO REALLY GOING TO MISS HER AND
ARE MISSING HER, AND WANTED TO
JUST ACKNOWLEDGE HER WONDERFUL
SERVICE TO THE INSTITUTE AND TO
NIH.
THANKS, BOB.
>>THANKS, CLINT.
JANICE HAS BEEN A REAL PILLAR OF
CLINICAL RESEARCH HERE AT THE
INSTITUTE, AND WILL BE MISSED.
OKAY.
GOODBYE AND CONGRATULATIONS TO
ALL OF YOU.
I JUST REALIZED THAT WE HAVE --
SPEAKING OF THE CONFUSION
BETWEEN HANKS ON COUNCIL, WE
ALSO HAVE TWO KEVINS HELPING US
WITH I.T., AND THE ONE SHOWING
THE SLIDE ACTUALLY I BELIEVE IS
KEVIN BISCANO, NOT KEVIN POWELL,
SO SORRY FOR THE FAUX PAS,
KEVIN.
THE LAST THING I WANT TO MENTION
IS WHAT'S CALLED OUR COUNCIL
OPERATING PROCEDURES, WHICH ARE
ESSENTIALLY THE GROUND RULES BY
WHICH WE INTERACT AT THE
INSTITUTE AND ON COUNCIL.
THIS YEAR WE USUALLY MAKE LITTLE
TWEAKS TO THEM EVERY YEAR OR SO.
WHAT WE'D LIKE TO DO THIS YEAR
IS TO MAKE A MINOR CHANGE TO
THESE DELEGATE AUTHORITIES.
SO THESE ARE THE ACTIONS THAT --
THE DELEGATED AUTHORITIES ARE
THE ACTIONS COUNCIL ALLOWS US TO
TAKE WITHOUT EXPLICIT COUNCIL
APPROVAL, FOR EXAMPLE, BECAUSE
THEY'RE TIME-SENSITIVE.
SO ONE EXAMPLE IS THAT WE'RE
ALLOWED TO GIVE ADMINISTRATIVE
SUPPLEMENTS TO GRANTS FOR UP TO
$100,000.
SO FOR EXAMPLE IF A LAB HAS A
CRISIS WHERE THEY LOSE A BIG
PIECE OF MACHINERY OR THEY LOSE
AN ANIMAL COLONY AND MAKE A
REASONABLY REQUEST, WE CAN
USUALLY TURN IT AROUND QUICKLY
BECAUSE WE DON'T HAVE TO WAIT
FOR A COUNCIL MEETING SO WE HAVE
A SMALL BUDGET FOR THAT.
AND IF THE REQUEST FOR A
SUPPLEMENT IS FOR MORE THAN
$100,000, WE BRING TO COUNCIL.
EITHER WE DISCUSS IT AT COUNCIL,
WE'LL ACTUALLY HAVE ONE EXAMPLE
TOMORROW, OR WE PUT IT ON THE
MISCELLANEOUS LIST AND WE REVIEW
IT THAT WAY THROUGH COUNCIL.
WHAT WE WANT TO ASK FOR TODAY IS
ESSENTIALLY AN ANALOGOUS
AUTHORITY FOR SMALL COFAs
GRANTS, PARTICULARLY GRANTS TO
OTHER INSTITUTES.
SO I'LL GIVE YOU AN EXAMPLE, IF
ANOTHER INSTITUTE IS FUNDING AN
R-13 GRANT, WHICH IS A
CONFERENCE GRANT, LET'S SAY IT'S
ON NEUROFIBROMATOSIS AND THEY
WANT COFUNDS FROM OTHER
INSTITUTES WHO HAVE A STAKE IN
THIS PARTICULAR AREA OF DISEASE
RESEARCH OR BASIC RESEARCH, WE
DO THAT BY CO-FUNDING ONE OF
THEIR GRANTS.
THE MONEY IS OFTEN USED TO BRING
IN NEUROSCIENCE SPEAKERS OR
NEUROLOGY SPEAKERS, AND THESE
SMALL COFUNDS ARE USUALLY
TIME-SENSITIVE.
WE HAVE TO SYNC WITH THE OTHER
INSTITUTE'S CALENDAR SO IT'S
VERY DIFFICULT TO WAIT FOR A
COUNCIL MEETING.
SO AGAIN, IF THE PROPOSED
CO-FUND EXCEEDS $100,000, WE
WOULD BRING TO COUNCIL TO GET IT
APPROVED.
SO ARE THERE ANY QUESTIONS ABOUT
THAT?
I DO NEED TO ASK FOR A FORMAL
MOTION TO APPROVE.
ARE THERE ANY QUESTIONS ABOUT
WHAT I'M TALKING ABOUT?
TIM?
>>NO, THAT WAS A MOTION TO
APPROVE WITHOUT QUESTIONS.
>>OKAY.
ANY -- ANY QUESTIONS ABOUT THIS
CO-FUNDING PROCESS?
WE DON'T DO IT ALL THAT OFTEN,
BUT IT'S USEFUL TO BE ABLE TO DO
IT QUICKLY WHEN WE NEED TO.
OKAY.
SO I'LL TAKE THAT MOTION, WHICH
I PRESUME IS TO APPROVE.
DO WE HAVE A SECOND FOR THAT?
AND THEN YOU CAN VOTE IN THE
CHAT.
SO THAT'S THE END OF OUR COUNCIL
DELEGATED AUTHORITIES.
JUST WANT TO MAKE SURE I DIDN'T
SKIP ANYTHING.
I THINK THAT'S EVERYTHING.
OKAY.
SO NOW WE HAVE WALTER'S TALK,
WHICH I UNDERSTAND IS
PRERECORDED, RIGHT, WALTER?
>>RIGHT.
WE'RE COMING FROM THE NEW
COMMAND CENTER HERE.
WE HAVE NEWLY RENOVATE SPACE AND
HOPEFULLY WE GOT YOU GUYS DOWN
HERE.
IT'S REALLY BEAUTIFUL NOW.
AND PEOPLE COMING IN IN DRIBS
AND DRABS BUT GOOD TO SEE PEOPLE
BACK AGAIN AFTER THOSE COUPLE
YEARS OF COVID.
AND SO THE DIRECTOR'S MESSAGE
HELPS ME PRACTICE AND MAKE SURE
IT'S SUCCINCT, RATHER THAN
AD-LIBBING IT, SO WHY DON'T WE
SHOW THAT.
WELL, MAYBE WE WON'T.
HOLD ON.
>>I WANT TO FIRST START OFF BY
ACKNOWLEDGING THE PASSING OF
RALPH SACCO.
RALPH WAS --
>>I THINK WE NEED TO TURN THE
SOUND WAY UP.
-- THE INSTITUTE WORK ON HEALTH
DISPARITIES FOR MUCH OF HIS
CAREER IN THE AREA OF STROKE.
HE WAS THE FIRST -- HELLO.
I'M WALTER KOROSHETZ, DIRECTOR
OF NINDS, AND I'M HAPPY TO GIVE
THIS DIRECTOR'S REPORT AND
WANTED TO THANK LINDY WOOD AND
AMY ADAMS FOR HELP WITH THIS
REPORT.
I WANT TO FIRST START OFF BY
ACKNOWLEDGING THE PASSING OF
RALPH SACCO.
RALPH WAS A COUNCILMEMBER,
TREMENDOUS FRIEND, GRANTEE OF
THE INSTITUTE, WORKED ON HEALTH
DISPARITIES FOR MUCH OF HIS
CAREER IN THE AREA OF STROKE.
HE WAS THE FIRST NEUROLOGIST TO
BE PRESIDENT OF THE AMERICAN
HEART ASSOCIATION, AND HE WAS
ALSO PRESIDENT OF THE ACADEMY OF
NEUROLOGY.
WE WILL REALLY MISS RALPH.
I WANT TO TALK TO A COUPLE OF
LEADERSHIP CHANGES HERE AT NIH.
WE MENTIONED DR. NINA SCHOR IS
NOW THE INTRAMURAL DIRECTOR OF
ALL OF NIH.
DR. RENEE WEGRZYN IS NOW THE
HEAD OF ARPA-A AND JONI RUTTER
IS NOW OFFICIALLY THE DIRECTOR
OF THE NATIONAL CENTER OF
ADVANCING TRANSLATIONAL
SCIENCES.
AS YOU PROBABLY KNOW DR. FAUCI
STEPPED DOWN, DR. AUCHINCLOSS IS
NOW ACTING DIRECTOR.
AT THE FOGARTY INSTITUTE, ROGER
GLASS WHO RAN THE INSTITUTE FOR
MANY DECADES AND WAS REALLY A
WONDERFUL SOURCE OF INFORMATION
ABOUT HOW TO DO RESEARCH
OVERSEAS, WE MADE MULTIPLE TRIPS
WITH HIM TO VARIOUS COUNTRIES TO
BUILD OUR GLOBAL HEALTH PROGRAM.
HE IS -- THAT INSTITUTE IS BEING
NOW RUN BY DR. PETER KILMARX,
THE ACTING DIRECTOR, AND IN THE
AREA OF THE COMMON FUND, THE
DIVISION OF PROGRAM
COORDINATION, PLANNING AND
STRATEGIC INITIATIVES, JIM
ANDERSON HAS LEFT AND NOW ROB
EISINGER RETURNED TO NIH TO BE
THE ACTING DIRECTOR.
THESE PROGRAMS ARE AMAZINGLY
INTERESTING, HIGH IMPACT
PROGRAMS THAT DO RESEARCH THAT
CUTS ACROSS ALL THE INSTITUTES.
NINDS IS INVOLVED IN MANY OF
THEM, AS YOU CAN SEE HERE, THE
ONES IN THE GREEN BOXES ARE ONES
THAT ARE CO-LED BY NINDS.
WE HAVE STAFF ON ALL THESE
PROJECTS THAT ARE LISTED ON THIS
SLIDE.
I WANT TO GO TO TALK ABOUT THE
BUDGET.
SO THE BUDGET WAS SIGNED BY THE
PRESIDENT, AND SO WE KNOW OUR FY
23 NUMBERS.
THE NINDS BASE, WHICH IS THE
FUNDING THAT COMES TO NIH FOR
ALL OUR BASE ACTIVITY, EXCLUDES
THINGS LIKE THE BRAIN
INITIATIVE, THE HEAL INITIATIVE,
MUCH OF THE MANAGEMENT OF
ALZHEIMER'S-RELATED DEMENTIA
RESEARCH WHERE THE FUNDS COME
FROM THE AGING INSTITUTE AND NOT
INCLUDED IN THE BASE.
OUR BASE IS INCREASED TO
2.167 BILLION, A 4% INCREASE.
TOTAL NINDS APPROPRIATION,
HOWEVER, WHEN YOU ADD IN THOSE
OTHER FIGURES THAT I MENTIONED
BRINGS OUR APPROPRIATION BUDGET
TO $2.8 BILLION, AND THAT'S A
TOTAL CHANGE OF 7.8%.
AS AN INCREASE.
IF ONE LOOKS FURTHER AT THE
BUDGET, HERE IS THE BASE AGAIN,
4% INCREASE.
THE BRAIN INITIATIVE MONEY IS
DIVIDED INTO TWO BUCKETS.
ONE IS THE BASE BUDGET AND THE
OTHER IS THE CURES.
OVERALL WE SAW AN 11% INCREASE,
BUT THAT WAS REFLECTED BY A BIG
INCREASE IN THE CURES ACT
FUNDING, WHICH IS TIME-LIMITED
MONEY, AND A BIG DROP IN THE
BASE BUDGET WHICH IS GENERALLY
SOMETHING THAT DOESN'T CHANGE
BUT IT DID THIS TIME
UNFORTUNATELY.
THE HEAL INITIATIVE BUDGET HAD A
3.7% INCREASE.
REMEMBER THIS IS HELPING TO END
ADDICTION LONG TERM, AND HERE AT
NINDS, WE SEE THE FUNDS AND IT
GOES ON TO PROJECTS THAT ARE
BEGUN BY MANY INSTITUTES TO
DEVELOP NON-ADDICTIVE AND MUCH
MORE EFFECTIVE TREATMENTS FOR
PAIN.
IN THE ALZHEIMER'S AND
ALZHEIMER'S DISEASE-RELATED
DEMENTIA AREA, FOR THE FIRST
TIME NINDS RECEIVED AN
APPROPRIATION OF $75 MILLION.
THIS IS FACTORED INTO THE FACT
THAT NINDS ADMINISTERS ABOUT 10%
OF THIS INITIATIVE FROM FUNDS
THAT ARE IN THE NATIONAL
INSTITUTE OF AGING, AND THE
TOTAL BUDGET FOR OUR PART OF THE
PROJECT IS $307.7 MILLION, NOW
$75 MILLION OF THAT WILL BE FROM
OUR APPROPRIATION.
NINDS IS ALSO THE LEAD IN THE
UNDIAGNOSED DISEASE NETWORK
CONTINUATION.
WE RECEIVED $2 MILLION TO OUR
BASE BUDGET FOR THAT PURPOSE.
THIS IS KIND OF A SUMMARY THEN
OF THE FUNDS THAT WE ARE
MANAGING, AND THE POINT TO MAKE
IS THAT THE FUNDS THAT WE'RE
MANAGING HAVE OVER DOUBLED
SINCE -- PRETTY MUCH DOUBLED
SINCE I BECAME DIRECTOR, AND
HATS OFF TO OUR PEOPLE WHO ARE
WORKING FURIOUSLY TO BE GOOD
STEWARDS OF THESE TACK PAIR
FUNDS.
CERTAINLY OUR PERSONNEL HAVE NOT
DOUBLED IN THAT TIME.
I WANT TO TALK A LITTLE BIT
ABOUT A PROBLEM THAT OCCURRED AT
THE NEUROSCIENCE RESEARCH
CENTER.
THOSE WHO MAY KNOW, IT'S A
FANTASTIC INSTITUTE HERE ON THE
NIH CAMPUS, HOME FOR
85 LABORATORIES, 800 SCIENTISTS
ALL IN THESE BUILDINGS THAT
FOSTER COLLABORATION, BUT JUST
BEFORE CHRISTMAS, WE HAD TWO
CATASTROPHIC FLOODS.
SOME OF THE HORRIBLE PICTURES.
THE WATER CAME POURING DOWN FROM
THE -- FROM REALLY THE ROOF OF
THE BUILDING AND THE TOP AREAS
OF THE BUILDING AND FLOODED ALL
THE LABORATORIES BELOW, CAUSING
REALLY CATASTROPHIC DAMAGE TO
SUPPLIES, EQUIPMENT.
THE LABS THAT ARE BADLY AFFECTED
ARE STILL NOT OPEN.
A LOT OF WORK GS ON IN T GOING S
OF REMEDIATION OF THE FLOOD
DAMAGE, AND THEN DEVELOPING A
SENSE OF THE EQUIPMENT AND
SUPPLY DAMAGES THAT NINDS WILL
HAVE TO REIMBURSE.
18 OF THE LABS THAT WERE DAMAGED
WERE NINDS LABS.
I WANT TO NOW TURN TO
AMYOTROPHIC LATERAL SCLEROSIS OR
A.L.S.
WE'RE GOING TO BE HEARING A LOT
ABOUT A.L.S. LATER IN THE OPEN
SESSION.
I JUST WANT TO BRING TO YOUR
ATTENTION THAT CONGRESS PASSED
AND THE PRESIDENT SIGNED THE
ACCELERATING ACCESS TO CRITICAL
THERAPIES A.L.S. ACT.
THAT ACT INCLUDES A COUPLE OF
COMPONENTS WHICH WE'LL TALK
ABOUT BRIEFLY HERE AND WE'LL
TALK ABOUT MORE EXTENSIVELY THIS
AFTERNOON.
THE FIRST IS THAT IT WAS MADE
VERY CLEAR THAT THE TOP PRIORITY
FOR THE FUNDS THAT CAME WITH
THIS BILL, NOW $75 MILLION IN
'23, ARE TO BE USED FOR EXPANDED
ACCESS RESEARCH.
SO THAT'S TO FUND GRANTS FOR
RESEARCH IN PERSONS WHO ARE
TREATED WITH INVESTIGATIONAL
INTERVENTIONS, WHO ARE NOT
ELIGIBLE FOR ONGOING PHASE
3 TRIALS, AND THESE AGENTS HAVE
TO COME FROM INDUSTRIES THAT
QUALIFY SMALL BUSINESS GRANTS.
IN FY '23, WE HAD $25 MILLION
THAT CAME TO NIH FOR THIS
PURPOSE.
WE SPENT $18 MILLION ON A
EXPANDED ACCESS RESEARCH GRANT,
AND THESE INCLUDE THE COSTS OF
MANUFACTURE AND DISTRIBUTION, IN
ADDITION TO THE USUAL COSTS THAT
WE SEE IN RESEARCH GRANTS.
THOUGH THE LIST OF ELIGIBLE
INTERVENTIONS, THEY HAVE TO BE
INTERVENTION FROM A SMALL
BUSINESS GRANT ORGANIZATION, AND
THEY HAVE TO BE IN PHASE
3 TRIAL, THE LIST IS LIMITED BUT
WE HOPE TO HAVE A ROBUST
RESPONSE TO THE FUNDING
ANNOUNCEMENT GIVEN THE LONGER
LEAD TIME AND THE OUTREACH FROM
NINDS AND THE A.L.S. COMMUNITY.
SO I'M ASKING ALL THOSE WHO ARE
INVOLVED IN A.L.S. TO MESSAGE
OUT THIS AMAZING RESEARCH
OPPORTUNITY, WHICH ALSO ALLOWS
MANY MORE PEOPLE WHO HAVE A.L.S.
TO RECEIVE INVESTIGATIONAL DRUGS
SHOULD THEY WISH TO DO SO.
THE FUNDING OPPORTUNITY WAS
RELEASED IN JANUARY, AS SOON AS
WE COULD GET IT OUT AFTER THE
BILL WAS -- AFTER THE BUDGET
CAME THROUGH.
WE HAVE TWO TECHNICAL WEBINARS
SCHEDULED.
I'LL SHOW YOU THAT ON THE NEXT
SLIDE.
NOW, THERE ARE $75 MILLION IN
THIS YEAR.
IF FUNDS ARE STILL AVAILABLE,
CONGRESS INSTRUCTED US TO USE
THEM FOR MERITORIOUS EXPANDED
ACCESS APPLICATIONS, BUT ALSO TO
EXPAND THE PUBLIC-PRIVATE
PARTNERSHIP FOR RARE
NEURODEGENERATIVE DISEASES
BETWEEN THE NIH AND FDA.
THIS WAS ALSO PART OF THE ACT IN
FY 22, WE TRANSFERRED FUNDS TO
THE FDA FOR A PUBLIC-PRIVATE
PARTNERSHIP RUN THROUGH THE
CRITICAL PATH INSTITUTE, AND
ALSO A NATURAL HISTORY STUDY OF
A.L.S. THAT WAS INSTITUTED BY
THE FDA.
IN '23, THE NINDS WILL PREPARE
FOR A POTENTIAL TO EXPAND THE
PUBLIC-PRIVATE PARTNERSHIP WITH
THE FDA AND C-PATH BY WORKING
WITH OUR PARTNERS AT THE
FOUNDATION FOR NIH.
A THIRD PRIORITY, SHOULD FUNDS
STILL BE AVAILABLE AFTER THE
PUBLIC-PRIVATE PARTNERSHIP AND
EXPANDED ACCESS IS FUNDED ARE
FUNDS FOR MERITORIOUS A.L.S.
RESEARCH.
THESE ARE THE LINKS TO THE FOA
AND ALSO LINKS TO THE WEBINARS
THAT WILL BE OCCURRING IN MID
AND LATE FEBRUARY TO GIVE
TECHNICAL ASSISTANCE WITH THOSE
WHO ARE INTERESTED IN APPLYING
FOR THE EXPANDED ACCESS RESEARCH
PROGRAM.
THANK YOU.
I JUST DID WANT TO MENTION THAT
A.L.S. RESEARCH HAS EXPANDED
DRAMATICALLY IN THE LAST FIVE TO
SIX YEARS.
THIS IS IN TERMS OF THE FUNDING
BUT ALSO THE LEVEL AND QUALITY
OF THE SCIENCE.
MUCH OF THESE FUNDS ARE FUNDS
THAT NINDS MANAGES THAT HAVE
COME FROM THE ADRD FUNDING THAT
WENT TO THE NATIONAL INSTITUTE
OF AGING AND WE MANAGE MOST OF
THE FUNDS RELATED TO
FRONTOTEMPORO DEMENTIA WHICH HAS
A SIGNIFICANT OVERLAP WITH
A.L.S.
YOU'LL HEAR ABOUT THE STRATEGIC
PLANNING THIS AFTERNOON, AND
YOU'LL HEAR FROM THE MEMBERS OF
THE STRATEGIC PLANNING GROUP,
RITA SATTLER, ROB BROWN, LAYNE
OLIFF, ELLEN PHILLIPS AND DAN
ALSO BEEN ASKED TO CARRY OUT A
STUDY OF PUBLIC AND PRIVATE
NON-PROFIT PARTNERSHIP HOW TO
MAKE ALZHEIMER'S A MORE LIVABLE
DISEASE WITHIN A DECADE, BUILD
UPON THE RESEARCH PRIORITIES IN
THE NIH A.L.S. STRATEGIC
PRIORITIES WITH A HEAVY EMPHASIS
ON CARE AND SERVICES.
IN THE SAME LINE AS
NEURODEGENERATIVE DISEASE
RESEARCH, WE OPENED THE ROY
BLUNT CENTER FOR ALZHEIMER'S
DISEASE AND RELATED DEMENTIAS
RESEARCH ON TH THE CAMPUS OF THE
NIH.
YOU CAN SEE ANDY SIN DELTON FROM
THE AGING INSTITUTE DIRECTOR,
SENATOR BLUNT, HIS WIFE,
DR. HODES AND I, CUTTING
RESEARCH ON THE NIH CAMPUS TO
DEVELOP BETTER YOU UNDERSTAND
STANDING OF THERAPIES FOR
ALZHEIMER'S AND THE ALZHEIMER'S
RELATED DEMENTIAS, WHICH THE
LATTER ARE DEFINED AS THE
VASCULAR CONTRIBUTION DEMENTIA,
FRONTOTEMPORO DEMENTIA AND LEWY
BODY DISEASE.
I WANTED TO BRING UP THE FACT
THAT NIH HAS BEEN LOOKING TO
MAKE SOME CHANGES IN ITS
RESEARCH PROJECT GRANT REVIEW
PROCESS.
THE IDEA IS HERE TO IDENTIFY THE
STRONGEST PRO TENSIONALLY
HIGHEST IMPACT RESEARCH BY
REDUCING THE BIAS IN REVIEW.
THERE'S SOMETHING CALLED THE
MATHEWS EFFECT, WHICH IS PEOPLE
WHO ARE REALLY FAMOUS AND PEOPLE
COME FROM FAMOUS INSTITUTIONS
TEND TO GET A BUMP IN THERREL --
IN HOW THEIR PROJECTS ARE
ASSESSED, AND THIS IS REALLY NOT
FAIR TO SOMEBODY WITH THE SAME
PROJECT, THE SAME EXPERTISE AND
THE SAME ABILITY AT ANOTHER
PLACE.
SO THE IDEA IS TO TRY TO
MINIMIZE THE INFLUENCE OF THE
APPLICANT'S RESUME AND
WORKPLACE.
SO WHAT THEY PROPOSE T TO DO ISO
HAVE BASICALLY THREE FACTORS NOW
TO ASK REVIEWERS TO SCORE.
THE FIRST TWO, AND THEN TO
INCORPORATE THE THIRD AS WELL AS
THE FIRST TWO INTO THE OVERALL
SCORE.
THE FIRST IS THE IMPORTANCE OF
RESEARCH, THE SECOND IS THE
FEASIBILITY AND THE RIGOR OF
THAT RESEARCH, AND THE THIRD ONE
IS THE EXPERTISE AND RESOURCES.
WE'RE REALLY TRYING TO SEPARATE
WHAT IS THE REAL EXPERTISE FROM
JUST THE REPUTATION THAT MAY NOT
ACTUALLY BEAR ON THE EXPERTISE
OR THE ENVIRONMENT ON THE
RESOURCES.
THERE WILL BE A REQUEST FOR
INFORMATION COMING ON -- I'M
SORRY, THERE IS A REQUEST FOR
INFORMATION OUT.
COMMENTS ARE ACCEPTED THROUGH
MARCH 10TH AT THIS WEBSITE.
TURNING NOW TO THE NIH DATA
MANAGEMENT SHARING POLICY, WHICH
WE'VE TALKED ABOUT MULTIPLE
TIMES, MULTIPLE COUNCILS, BUT
THE MAIN POINT IS APPLICATIONS
RECEIVED AFTER JANUARY 25TH,
2023 MUST INCLUDE A PLAN FOR
SHARING BOTH PUBLISHED AND
NON-PUBLISHED SCIENTIFIC DATA.
SO THIS IS NOW IN EFFECT.
THERE IS A GREAT DEAL OF
GUIDANCE AVAILABLE FOR PEOPLE TO
UNDERSTAND HOW THEY CAN COMPLY
WITH THIS MANDATE FROM NIH.
MANY PLACES TO POST THE DATA.
WE WELCOME YOUR FEEDBACK ON AN
ONGOING BASIS BUT I'VE BEEN
URGING THE COMMUNITY TO PIVOT TO
THINKING NOT JUST HOW TO POST
THE DATA BUT REALLY HOW DO WE
MAKE BEST USE OF THIS POSTED
DATA SO THAT'S GOING TO ADVANCE
YOUR FIELD OF SCIENCE.
AND I THINK THAT'S GOING TO TAKE
WORK, BUT I THINK THAT'S WHERE
THE BENEFIT IS GOING TO BE.
IN A SIMILAR VEIN, I WANTED TO
BRING UP THE NEW POLICY COMING
OUT OF THE WHITE HOUSE WITH
REGARD TO PUBLIC ACCESS TO
FEDERALLY FUNDED DATA.
SO THE CURRENT POLICY IS THAT
THE DATA THAT IS PUBLISHED,
FUNDED BY NIH HAS TO BE MADE
PUBLICLY AVAILABLE NO LATER THAN
12 MONTHS AFTER THE DATE OF
PUBLICATION.
BUT THE NEW GUIDANCE IS FOR THAT
TO BE MADE IMMEDIATELY
AVAILABLE.
SO NIH IS PUTTING IN THEIR
PLANS, THEY WOULD DO -- I THINK
THEY WERE DUE ALREADY, AND THESE
POLICIES WOULD HAVE TO GO INTO
EFFECT AS OF DECEMBER 31ST,
2025.
SO STAY TUNED.
A DRAFT PLAN AND OPPORTUNITY FOR
PUBLIC COMMENT ON HOW NIH WILL
RESPOND TO THE WHITE HOUSE
MEMORANDUM HERE WILL BE COMING
OUT.
WE'RE GOING TO TALK ABOUT THE
ADVISORY COMMITTEE TO THE
DIRECTOR.
THESE COMMITTEES OF THE
DIRECTOR'S COUNCIL, AND THEY ARE
VERY MUCH UTILIZED TO DEVELOP
POLICIES THAT CUT ACROSS ALL THE
INSTITUTES.
ONE HAS SUBMITTED ITS REPORT AND
THAT'S THE GROUP ON DIVERSITY
AND INDIVIDUALS WITH
DISABILITIES.
THEIR REPORT INDICATES AND TELLS
US THAT PERSONS WITH
DISABILITIES ARE THE SINGLE
LARGEST MINORITY POPULATION AND
FASTEST GROWING MINORITY
POPULATION IN THE U.S.
THEY REPRESENT ABOUT 9% OF THE
SCIENTIFIC WORKFORCE.
AND THE ITION INDICATION IS
DISABILITIES I -- DISABILITY
REPRESENTATION IN THE SCIENTIFIC
WORKFORCE DECREASES THROUGHOUT
THE CAREER PATH, ACCOMMODATE
PEOPLE FOR DISABILITIES AND TO
EP ABLE THEM TO FUNCTION AT THE
HIGHEST LEVEL AT NIH
ENTERPRISES.
SUGGESTIONS FOR THE ACD --
COMING OUT OF THE ACD INCLUDED
UPDATING THE NIH MISSION
STATEMENT.
THAT MISSION STATEMENT IS TO
SEEK FUNDAMENTAL KNOWLEDGE ABOUT
THE NATURE AND BEHAVIOR OF
LIVING SYSTEMS AND THE
APPLICATION OF THAT KNOWLEDGE TO
ENHANCE HEALTH, LENGTHEN LIFE,
AND REDUCE ILLNESS AND
DISABILITY, AND THIS IS
CONSIDERED, YOU KNOW, A SIGN OF
ABLEISM AND THE NINDS MISSION
STATEMENT DOES NOT INCLUDE THE
TERM DISABILITY, IT'S REALLY TO
REDUCE THE BURDEN OF ILLNESS,
AND EVEN THAT, WE'RE THINKING OF
CHANGING IN CASE IT'S
INTERPRETED AS THE BURDEN FOR
WHO.
IF IT'S THE BURDEN OF SOCIETY,
THAT'S NOT OUR PLAN.
OUR PLAN IS REDUCE THE BURDEN
FOR THE INDIVIDUALS WHO ARE
SUFFERING.
SO WE'RE CONSIDERING HOW TO DEAL
WITH THAT ISSUE OURSELVES.
IIT ASKS US TO DEVELOP AN NIH
WIDE EFFORT TO IDENTIFY AND
ADDRESS ANY STRUCTURAL ABLEISM
THAT MAY EXIST AND ASK US TO
PROMOTE DISABILITY INCLUSION
INTERNALLY AND AT THE NIH FUNDED
EXTRAMURAL WORKFORCE.
EXPAND EFFORTS TO INCLUDE
DISABILITY COMMUNITIES AND THEIR
PERSPECTIVES.
AND CONDUCT RESEARCH ON HEALTH
DISPARITIES AFFECTING THOSE
PEOPLE WITH DISABILITIES.
AND ENSURE THAT DISABILITY
INCLUSION AND ANTI-ABLEISM ARE
CORE COMPONENTS OF ALL DEI
EFFORTS.
OF INTEREST, THE NINDS HAS I
THINK REALLY BEEN AT THE
FOREFRONT.
OUR FOLKS PUT TOGETHER A
STRATEGIC PLAN WITH CORE IDEALS
TO MAKE US A REALLY WELCOMING
COMMUNITY TO WORK IN FOR
EVERYONE.
AND ONE OF THE THINGS WE DO TO
DO THAT IS TO HOLD COMMUNITY
CONVERSATIONS WHICH ARE LIKE
TOWN HALLS ON DIFFERENT TOPICS,
SOMETIMES WITH PRESENTATIONS,
AND YOU CAN SEE THE KIND OF
THINGS WE TALKED ABOUT, MICRO
AGGRESSIONS, ANTI-ASIAN BIAS,
AND THEN WE HAD ONE OCTOBER 14TH
WHICH WAS REALLY FANTASTIC ON
UNDERSTANDING THE ISSUES FACED
BY PEOPLE WHO DO HAVE
DISABILITIES, AND SO THIS IS
JUST A COUPLE THINGS TAKEN OUT,
WE HAD A GREAT TURNOUT, 485, 88%
OF OUR STAFF ATTENDED THIS
CONVERSATION.
AND 7%, WE SAID 9% OF THE
WORKFORCE FOR US IT'S 7% OF
THOSE RESPONDING INDICATED THEY
DID HAVE A DISABILITY.
ABOUT 5 DID NOT IDENTIFY YES OR
NO.
AND THE DISCUSSIONS WERE REALLY
POIGNANT.
PEOPLE TALKED ABOUT TROUBLES
THAT THEY'VE HAD, GIVEN THEIR
DISABILITIES, AND ALSO
SUGGESTIONS ABOUT HOW THINGS
COULD BE SO MUCH BETTER, AND SO
WE'RE TAKING THOSE TO HEART TO
MAKE OUR INSTITUTION A REALLY
FANTASTIC PLACE TO WORK FOR
EVERYONE.
THE NEXT ADVISORY COMMITTEE TO
THE DIRECTOR, I WANTED TO
MENTION, IS LOOKING AT THE ISSUE
ABOUT DECLINE IN POSTDOCTORAL
STUDENTS AND TRAINEES.
SO YOU MAY HAVE SEEN IN THE
LITERATURE THE CASE OF THE
DISAPPEARING POSTDOCS.
I CERTAINLY IN EVERY KIND OF
CONVERSATION THAT I'VE HAD WITH
RESEARCHERS AND RESEARCH
INSTITUTIONS, THIS COMES UP AS
A -- ALMOST NUMBER ONE PROBLEM,
DECLINE IN THE NUMBER OF
POSTDOCS.
SO NIH CAN PUT OUT MORE FUNDING,
OFTEN TIME THAT WORK IS DONE
BEEN THE POSTDOCS, SO THIS IS A
TREMENDOUS EFFECT ON WHAT WE CAN
GET DONE WITH OUR FUNDING AS
WELL.
AS WELL AS THE FACT THAT WE'RE
TRYING TO HELP PEOPLE DEVELOP
ACADEMIC CAREERS OR CAREERS IN
INDUSTRY THAT ARE GOING TO
ADVANCE OUR MISSION.
SO THE COMMITTEE WILL EVALUATE
THE PERCEIVED SHORTAGE OF
PH.D.s SEEKING POSTDOCS.
LOOK AT THE FACTORS UNDERLYING
THIS, COMPARE DIFFERENT
APPROACHES TO POSTDOCTORAL
TRAINING, FIGURING OUT WAYS TO
BETTER IMPROVE THE QUALITY OF
LIFE/WORK BALANCE AND INCREASE
RETENTION IN SCIENCE AT THIS
POSTDOC LEVEL.
THESE ARE THE MEMBERS OF THE
COMMITTEE.
NOTE THAT NINA SCHOR, NOW THE
DIRECTOR OF INTRAMURAL, IS ON
THE COMMITTEE, AND HERE IS A
COUPLE OF NUMBERS.
THIS IS I THINK NATIONAL SCIENCE
FOUNDATION DATA ON THE NUMBER OF
POSTDOCS OVER TIME.
YOU CAN SEE THIS DECLINE THAT
OCCURRED STARTING AROUND 2010,
MAYBE A LITTLE PICKUP, BUT THEN
ANOTHER DECLINE.
THIS IS THE DECLINE OF POSTDOCS
SUPPORTED ON NIH TRAINING
GRANTS, SO WE'RE SEEING LOWER
POSTDOC NUMBERS OVERALL, FEWER
F32, THESE ARE THE POSTDOC
APPLICATIONS AND AWARDS.
THE STIPENDS HAVE INCREASED BUT
NOT PACING INDUSTRY.
THE NUMBER OF GRAD STUDENTS
PLANNING ACADEMIC POSTDOCS
CONTINUES DOWNWARD TREND.
GRAD STUDENTS AND POSTDOCS ARE
REPORTING DOWNWARD TREND IN THE
QUALITY OF LIFE, AND THAT'S
WORSE REPORTS FROM INTERNATIONAL
TRAINEES.
AND ANECDOTAL DISCUSSIONS LIKE
THE ONES I MENTIONED, WE'VE HAD
SAY WITH SOCIETY FOR
NEUROSCIENCE, SUGGESTS THE
TRENDS ARE WORSENING SINCE 2020.
AT NINDS, HERE'S OUR DATA.
YOU CAN SEE THE F32s IN THE
LIGHT GREEN HAVE BEEN DECLINING
SIGNIFICANTLY OVER THE LAST FIVE
TO SIX YEARS.
THIS HAS BEEN SOMEWHAT BALANCED
BY AN INCREASE IN THE NUMBER OF
K99 APPLICATIONS AWARDED AND
RECEIVED.
THE K99s ARE ACTUALLY FUNDING
PEOPLE TO DO WORK IN THE END OF
THEIR POSTDOC AND THEN
TRANSITION THEM TO A PERMANENT
POSITION IN THE R00 PHASE.
SO THOSE ARE ALSO INCREDIBLY
VALUABLE, THAT'S WHAT REALLY
GETS PEOPLE INTO THE JOBS, BUT
WE NEED THAT WORKFORCE --
POSTDOC WORKFORCE UNDERNEATH TO
GET TO THAT LEVEL.
NOW THE GOOD NEWS IS THAT EARLY
STAGE INVESTIGATORS, AGAIN,
PEOPLE WHO JUST GOT THEIR FIRST
JOB AND THIS IS THEIR FIRST
GRANT, THOSE NUMBERS ARE GOING
UP ACROSS NIH AND ACROSS NINDS.
I THINK WE WERE A LITTLE BIT
AHEAD OF THE CURVE YEARS AGO,
AND NOW WE'RE JUST STILL A
LITTLE BIT ABOVE, WE FUND ABOUT
7% OF THE NIH ESIs IN '22, AND
WE'RE ABOUT 6% OF THE NIH
BUDGET.
SO EARLY STAGE INVESTIGATORS ARE
BEING FUNDED, WHICH MEANS JOBS
ARE OPENING UP FOR THESE NEW
PEOPLE, AND THIS K99 IN THE
EARLY STAGE INVESTIGATIVE BUMP,
WE FUND UP TO THE 25 PERCENTILE
FOR EARLY STAGE INVESTIGATORS, I
THINK IS REALLY HELPING TO GET
THESE JOBS OPEN.
THERE'S ANOTHER ADVISORY
COMMITTEE TO THE DIRECTOR GROUP
I WANTED TO BRING YOUR ATTENTION
TO, AND THIS GROUP IS LOOKING AT
HOW TO CATALYZE THE DEVELOPMENT
AND THE USE OF NOVEL,
NON-ANIMAL-BASED APPROACHES TO
ADVANCE BIOMEDICAL RESEARCH.
SO THEY'RE LOOKING AT
ALTERNATIVE METHODS BEING
DEVELOPED IN BIOMEDICAL RESEARCH
TO ASSESS THE STRENGTHS AND
WEAKNESSES FOR STUDYING HUMAN
BIOLOGY, CIRCUITS, SYSTEMS AND
DISEASE SYSTEMS.
NIMH -- I'M SORRY, NINDS AND DRD
THAT THESE METHODS WOULD MOST
LIKELY BE CONSIDERED
COMPLEMENTARY TO NEUROSCIENCE
RESEARCH THAT CAN ONLY BE
PERFORMED IN ANIMALS AND HUMANS.
IT'S HARD FOR US TO IMAGINE HOW
THE TECHNOLOGIES NOW AVAILABLE
ARE ACTUALLY ALTERNATIVES, BUT
CERTAINLY COMPLEMENTARY.
AND THESE ARE THE MEMBERS OF
THAT COMMITTEE, AND YOU REMEMBER
ARNOLD KRIEGSTEIN, NINDS
COUNCILMEMBER RECENTLY, IS PART
OF THAT GROUP.
JUST TO END UP, I WANTED TO SAY
THAT AS I MENTIONED IN THE
BEGINNING, OUR BUDGET HAS
INCREASED, WE HAVE GREAT NEED
FOR PEOPLE WHO WANT TO COME IN
AND MAKE A NATIONAL IMPACT ON
NEUROSCIENCE RESEARCH, AND SO WE
ARE HIRING, WE'RE LOOKING FOR
PEOPLE AND PROGRAM DIRECTOR,
PROGRAM ANALYST IN MANY OF OUR
DIFFERENT DIVISIONS, AND ALL
BECAUSE THE GROWTH OF THE
RESEARCH THAT -- BECAUSE OF THE
GROWTH OF THE RESEARCH THAT NOW
WE ARE STEWARDING.
AND JUST TO END UP BY SAYING
THAT THERE'S BEEN LOTS OF
PROGRESS EACH YEAR AT THIS TIME.
I TRY AND JUST THROW OUT A
COUPLE OF TIDBITS, BUT THESE ARE
TIDBITS -- THESE ARE TIPS OF THE
ICEBERG FOR ALL THE FANTASTIC
SCIENCE RESEARCH THAT'S GONE ON
ACROSS THE COUNTRY IN THE LAST
YEAR.
THESE ARE A COUPLE OF EXAMPLES
THAT WERE IN MY DIRECTOR'S
MESSAGE CALLED REFLECTING BACK
ON 2022, AND I CAN'T GO THROUGH
ALL OF THEM NOW, I'D LOVE TO,
BUT IT WOULD PROBABLY TAKE TOO
MUCH TIME, BUT THESE HAVE BEEN
REALLY FANTASTIC ADVANCES IN
MANY DIFFERENT ASPECTS OF
NEUROSCIENCE AND NEUROLOGICAL
DISORDERS.
SO WITH THAT, I WANT TO THANK
YOU FOR YOUR ATTENTION, HAPPY TO
ANSWER ANY QUESTIONS.
HAPPY TO ANSWER ANY QUESTIONS IF
ANYBODY WANTED TO BRING ANYTHING
UP?
GINA, YOU MUST HAVE SOMETHING.
>>YEAH, I'VE GOT A FEW.
OKAY.
SO THE BUDGET HAS ROUGHLY
DOUBLED SINCE YOU HAVE BECOME
DIRECTOR, AND YET THE PERSONNEL
HAVEN'T.
I THINK THAT THAT MIGHT MEAN
PROBABLY THAT THE PERSONNEL
EXISTING AT NINDS ARE OVERTAXED,
ESPECIALLY WITH TOO MANY GRANTS
TO HANDLE AND NOT ENOUGH PROGRAM
OFFICERS AND PROGRAM ANALYSTS.
I KNOW YOU WERE HIRING, THAT'S
ONE OF THE LAST THINGS YOU SAID,
THAT'S WONDERFUL.
IS THERE A WAY TO SHIFT AROUND
THE NUMBERS SO YOU CAN ACTUALLY
HIRE ENOUGH TO BE -- TO SERVICE
THE GRANTS AND THE RESEARCHERS
AS WELL AS THEY USED TO?
IS THAT THE PLAN?
>>OH, ABSOLUTELY.
MY UNDERSTANDING IS THAT THEY
ARE AS RESPONSIVE AS THEY ALWAYS
WERE.
THEY MIGHT BE A LITTLE BIT MORE
UNDER THE PRESSURE, BUT IF
THAT'S NOT THE CASE, WE'D LIKE
TO KNOW ABOUT IT.
RESPONSIVENESS FOR SURE, THAT
ALWAYS COMES FIRST.
>>IT MIGHT BE THAT, YOU KNOW,
THE PEOPLE IN THE EMPLOYEE --
EMPLOYMENT, UNDER EMPLOYMENT OF
NINDS AREN'T COMPLAINING BUT IT
ALSO MIGHT BE THAT THEY ARE
SUPER STRESSED AND IT MIGHT
BE -- MAYBE WE CAN DO AN
INTERNAL COMMITTEE THAT STUDIES
THE HEALTH AND WELLNESS AND
TURNOVER OF CURRENT STAFF JUST
TO MAKE SURE THAT IT IS INDEED
HEALTHY AND WE ARE HIRING AT THE
APPROPRIATE LEVELS, MY
SUGGESTION.
>>THANKS FOR THAT, GINA.
YEAH, I THINK THEY ARE
OVERSTRESSED, NO QUESTION ABOUT
IT.
IT'S NOT GOOD FOR THE HEALTH OF
THE GROUP, NO QUESTION ABOUT IT.
AND WE'VE BEEN TRYING EVERYTHING
WE CAN TO BRING IN PEOPLE.
WE'VE HAD SOME SUCCESS, BUT THE
REASON I BROUGHT IT UP IS WE
REALLY NEED HELP.
IT'S A GREAT JOB, PEOPLE DON'T
KNOW ABOUT IT ON THE OUTSIDE,
BUT WE HAVE I DON'T KNOW HOW
MANY VACANCIES OPEN.
>>28HSAs RIGHT NOW.
>>WHAT'S THAT?
>>28.
>>WOW!
THAT'S A LOT.
SO ARE WE PAYING ENOUGH?
IS THAT WHY?
OR IS THERE A BETTER WAY TO GET
THE WORD OUT FOR THE JOBS THAT
ARE AVAILABLE?
>>EXACTLY RIGHT, WE NEED TO GET
THE WORD OUT.
THE PAY, IT'S GS SCALE PAY, SO
THAT IS A PROBLEM MY SON IS A
LAWYER IN ENERGY AND HE GETS THE
SAME PAY.
SO IT'S SOMETHING YOU COME INTO
THE FEDERAL GOVERNMENT, YOU HAVE
STABILITY --
>>RETIREMENT.
>>BUT THE TOP PAY IS WHAT NOW?
GS15 IS --
>>ABOUT 175,000, I BELIEVE.
>>VERY TOP.
AND ON AVERAGE, MAYBE IT'S
SOMETHING ELSE.
AND YOU KNOW, I DON'T KNOW ABOUT
THE COST OF LIVING IN BETHESDA,
BUT, YOU KNOW, IT MIGHT ALSO BE
THAT, YOU KNOW, PEOPLE DON'T
HAVE TO LIVE THERE.
THEY CAN LIVE IN OTHER PLACES.
>>YES, WE ARE DOING THAT.
WE HAVE HIRED A NUMBER OF PEOPLE
WHO DO NOT LIVE IN D.C. .
WE ACTUALLY HAVE A NUMBER OF
PEOPLE WHO MOVED OUT OF D.C.
DURING COVID.
SO WE ARE TRYING TO PUT TOGETHER
THIS HYBRID WORK SPACE NOW WITH
PEOPLE WHO ARE REMOTE AND PEOPLE
WHO ARE ON CAMPUS.
WE REALLY NEED MORE PEOPLE GIVEN
THE DOUBLING OF THE BUDGET.
>>GINA, CAN YOU HEAR THE SOUND
OF OUR STAFF APPLAUDING IN THE
BACKGROUND?
>>I CAN'T, BUT I CAN IMAGINE.
IT JUST SEEMS LIKE, THE PEOPLE
WHO ARE THERE ARE DOING HEROS'
WORK AND I APPLAUD ALL OF YOU.
I WOULD JUST LOVE TO SEE MORE
SUPPORT, ESPECIALLY GIVEN THE
DOUBLING OF THE BUDGET OVER THE
YEARS.
>>YOU DON'T HAVE TO WORRY ABOUT
PEOPLE NOT MAKING THEIR STRESS
KNOWN.
THEY DEFINITELY ARE.
>>WELL, GOOD.
GOOD.
BUT MAYBE THERE SHOULD BE A
CONCERTED EFFORT OR A COMMITTEE
THAT ACTUALLY ASKS THE HARD
QUESTIONS.
>>YES, WE ACTUALLY HAVE EVEN
CONSIDERED HIRING A CONTRACTOR
TO REALLY LOOK AT THIS.
WE ARE FULL-FLEDGED LOOKING INTO
THIS.
>>GREAT.
>>I SEE KEN HAS A QUESTION.
>>THANK YOU.
SO I ACTUALLY -- JUST A QUICK
FOLLOW-UP FROM THE POINT THAT
GINA JUST RAISED.
I'M ASSUMING THAT EXIT
INTERVIEWS ARE CONDUCTED,
BECAUSE THAT'S THE KIND OF
INFORMATION THAT MIGHT HELP YOU
GET TO ROOT CAUSE.
YOU KNOW, SALARY IS ALWAYS --
SURE, ABSOLUTELY, BUT THERE
COULD BE OTHER THINGS.
AND BY DOING EXIT INTERVIEWS,
ESPECIALLY IF YOU HAVE PEOPLE
HEADING FOR THE HILLS IN ONE
PARTICULAR AREA OF YOUR
INSTITUTE, IT MIGHT HELP.
>>AMY?
>>I DO WANT TO JUST SAY THAT I
THINK -- THAT'S AN IMPORTANT
POINT AND WE DO DO EXIT
INTERVIEWS BUT I WANT TO ALSO
NOTE THAT OUR BIGGEST CHALLENGE
RIGHT NOW IS RECRUITMENT.
RETENTION IS ABSOLUTELY
IMPORTANT, AND SOMETHING FOR US
TO FOCUS ON.
WE HAVE INCREDIBLE STAFF AND WE
WANT TO KEEP THEM AND KEEP THEM
HAPPY IN THEIR JOB.
BUT THE REAL PAIN POINT RIGHT
NOW, AND IT IS ACUTE, IS
RECRUITING.
AND SO IN ADDITION TO ALL OF THE
IMPORTANT THINGS THAT WERE JUST
NOTED, ANY HELP THAT YOU CAN
GIVE US IN IDENTIFYING PEOPLE
THAT COULD CONTRIBUTE IN THIS
REALLY IMPORTANT AND MEANINGFUL
SPACE, WE WOULD WELCOME.
>>I WANT TO KEEP THIS MOVING
ALONG.
KEN?
>>SURE, SO I'LL MAKE THE
COMMENT QUICK.
IT IS ACTUALLY JUST TO
CONGRATULATE NIH FOR, YOU KNOW,
DOING SOMETHING TO LEVEL THE
PLAYING FIELD IN THE GRANT
REVIEW SPACE.
YOU KNOW, I THINK THAT NIH BUT
ALSO PRIVATE FOUNDATIONS HAVE
STRUGGLED FOR YEARS TO TRY AND,
YOU KNOW, ELIMINATE THE BIG NAME
IVY LEAGUE, YOU KNOW,
INVESTIGATOR WHO'S HAD 20 R01s
TO ELIMINATE THAT KIND OF THING,
AND I THINK THAT THIS APPROACH
WILL DO A LOT TO ENHANCE NIH'S
EMPHASEMPHASIS ALSO ON DIVERSIT,
EQUITY AND INCLUSION, BECAUSE I
THINK WE ALL KNOW THAT WHEN YOU
HAVE DIVERSITY OF THOUGHT,
INNOVATION GOES UP, PRODUCTIVITY
DPRPRODUCTIVITYGOES UP, SO JUST
CONGRATULATIONS.
>>THANKS A LOT, KEN.
TIM?
>>I HAD A QUESTION ABOUT THE
POSTDOC TRENDS WHICH WE'RE
ALL -- I'M SURE EVERYONE IS
ACUTELY AWARE OF, BUT THE DATA
CLEARLY LOOKED LIKE, WALTER,
FROM YOUR TALK, JUST FROM THE
F-32 APPLICANTS, THAT'S BEEN
GOING DOWN RATHER STEADILY, BUT
YET THE ESIs ARE AT LEAST
LEVEL.
THAT IMPLIES THAT THE PEOPLE
THAT WE'RE NO LONGER RECRUITING
ARE NOT THE ONES THAT WANTED
ACADEMIC JOBS.
THAT MUST BE STAYING CONSTANT IF
WE'RE STILL FEEDING ESIs,
RIGHT?
YET THE ONES WE'RE MISSING PLUB
PEOPLE THAT HAVE NO INTENTION OF
ACTUALLY -- OR NOT NO INTENTION
BUT IN THE END, THE ONES THAT
REALLY WANT TO GO ON ARE GOING
ON, WE'RE GETTING THOSE PEOPLE
AS POSTDOCS.
WE'RE MISSING THIS OTHER POOL.
I REALIZE WE NEED THEM TO DO THE
WORK, THEY DON'T ALL HAVE TO GO
ON TO ACADEMIA, BUT IT SUGGESTS
THAT AS ONE DIE SECRETARIES THIS
PROBLEDISSECTS THISPROBLEM, THEE
WE'RE NOT THINKING ABOUT.
>>MICHELLE JONES LONDON DID
WHAT'S CALLED THE Y SURVEY AND
THAT'S PUBLISHED IN NERURO,
THAT'S ONE REALLY GOOD EXAMPLE
OF WHY PEOPLE DECIDE TO LEAVE.
A LOT OF GOOD PEOPLE DECIDE TO
LEAVE FOR VARIOUS REASONS.
SO WE'RE MISSING -- WE'RE
STARTING OFF WITH TALENT, AND A
LOT OF THAT TALENT IS DRIPPING
AWAY AND LEAKING OUT.
AND WH ONE OF THE BIGGEST IS IN
WOMEN WHO NOW MAKE UP MORE THAN
50% OF THE GRADUATE STUDENTS AND
POSTDOCS, THAT THE LEAK OUT OF
ACADEMIA OCCURS WHEN THEY START
TO HAVE CHILDREN.
SO THAT IS SOMETHING THAT HAS
NOTHING TO DO WITH WHETHER THEY
WANT TO BE GREAT SCIENTISTS.
IT'S JUST, LIFE GETS IN THE WAY
AND WE DON'T HAVE THE RIGHT
ACCOMMODATION FOR THOSE, YOU
KNOW --
>>I FULLY -- THAT POINT HAS
BEEN MADE REALLY EFFECTIVELY FOR
A LONG TIME.
MY POINT IS THAT THE ESIs HAVE
NOT BEEN PLUMMETING AT THE SAME
RATE AT F-32s, WHICH MEANS
THAT WE'RE STILL FEEDING
ACADEMIA, I'M NOT TALKING ABOUT
THE GENDER BALANCE.
THAT HASN'T CHANGED, I AGREE.
BUT THERE'S SOMETHING UNUSUAL
GOING ON THAT WE'RE STILL
FEEDING -- IF IT WAS JUST
PROPORTIONAL, I'D SAY OKAY,
WE'RE NOT GETTING ENOUGH
POSTDOCS, WE'RE NOT PRODUCING
ENOUGH GOING INTO ACADEMIA
TOGETHER BUT WE ARE APPARENTLY
FILLING THE ACADEMIC JOBS.
>>WELL, I ACTUALLY WANT TO TALK
ABOUT THAT LAST POINT THAT
WALTER SAID, WHICH IS HAS BEEN A
PROBLEM FOR A LONG TIME.
WHEN WOMEN HAVE CHILDREN,
THEY'THEYNO LONGER VIEW A SCIENC
ACADEMIC JOB AS A VIABLE JOB,
AND WHY IS THAT?
THAT ALSO REQUIRES THAT WE
SHOULD TAKE A HARD LOOK AT THE
CULTURE OF ACADEMIC RESEARCH AND
FIGURE OUT WAYS TO MAKE IT GOOD.
WHY SHOULD THIS SELECTIVELY
IMPACT WOMEN?
IT SHOULDN'T.
YOU KNOW, IT SHOULD AFFECT WOMEN
AND MEN EQUALLY, BUT IT DOESN'T,
AND WHY IS THAT THE CASE?
WHAT ARE WE NOT DOING TO
MAINTAIN AND RETAIN THIS
AMAZING, BEAUTIFUL, YOU KNOW,
INTELLECTUAL RESOURCE OF WOMEN
IN ACADEMIA.
AND IT A SERIOUS PROBLEM.
>>HANK?
>>I MAY TAKE US AWAY FROM THIS
IMPORTANT DISCUSSION BUT IT'S A
SIMPLE POINT OF CLARIFICATION,
WALTER.
YOU MENTIONED THAT 10% OF THE
ALZHEIMER'S INITIATIVE IS
ADMINISTERED BY NINDS, AND THIS
YEAR YOU HAVE $75 MILLION.
THE QUESTION FROM ME IS, IS THAT
PART OF THAT 10% OR ABOVE AND
BEYOND THAT 10%?
>>IT WILL BE PART OF THAT 10%.
>>I'M SORRY, ARE YOU TALKING
ABOUT THE A.L.S. MONEY, HANK?
>>NO, THIS WAS ADRD.
>>OH, ADRD.
YEAH, THAT'S PART OF IT.
>>BECAUSE WE GOT 75, BUT WE
ACTUALLY MANAGE OVER
300 MILLION.
BUT WE'RE NOT SECEDING FROM THE
UNION.
>>SAMEER?
>>THANKS, WALTER.
SIMILAR TOPIC.
REGARDING BUDGET.
TYPICALLY WE SEE THE SORT OF
SLOW, STEADY INCREASE, AND
OBVIOUSLY WE CAN'T EXPECT IT BUT
IT HAS BEEN CONSISTENT OVER
YEARS.
THE BRAIN-BASED, YOU KNOW, TOOK
A BIG DECREASE THIS PAST BUDGET
CYCLE, SO I WONDER IF YOU HAVE A
SENSE OF READING THE TEA LEAVES
AS TO WHAT THAT MEANS, IS THAT
BALANCING SOMETHING ELSE OUT OR
IS THIS AN INDICATOR OF SOME
OTHER DISTRIBUTION OR PLAN?
>>I MEAN, THE TEA LEAVES ARE
HARD TO READ, BUT AS YOU KNOW,
THINGS HAVE CHANGED DRAMATICALLY
IN THE CONGRESS.
AND IT HARD TO IMAGINE ANY OF
THOSE CHANGES ARE GOING TO BE
BENEFICIAL FOR NIH.
LET'S JUST PUT IT THAT WAY.
WE COULD BE WRONG, BUT WHAT WE
DO EVERY YEAR IS PREPARE FOR A
FLAT BUDGET, AND I WOULD SAY
THIS YEAR MORE THAN EVER, WE'RE
GOING TO HAVE TO PREPARE FOR A
FLAT BUDGET.
SO I'M NOT SURE I CAN SAY MUCH
MORE THAN THAT, BUT I THINK WE
HAVE TO PREPARE FOR A FLAT
BUDGET.
>>THANKS.
>>GINA.
>>I WANTED TO CONGRATULATE YOU
ON A FANTASTIC ADDRESS BECAUSE
IT WAS SO MEATY AND INSPIRED SO
MANY QUESTIONS ON MY PART, BUT
ONE OF THEM IS THAT BLIND
REVIEW, OR THE POSSIBILITY OF
BLIND REVIEW.
I LIKE THE IDEA THAT WE'RE
SEPARATING OUT INSTITUTION AND
INVESTIGATOR REPUTATION, BUT I
DON'T THINK THAT'S GOING TO BE
ENOUGH TO IMPACT PEOPLE'S SCORES
AND TO CHANGE THE BIAS THAT
REPUTATION AND INSTITUTION GIVE
US, SO I WANTED TO SUGGEST THE
POSSIBILITY AGAIN OF ACTUAL
BLIND REVIEWS, JUST LIKE THEY
DID IN ASTRONOMY, OF TAKING OUT
ALL REFERENCE TO THE INSTITUTION
FROM THE REVIEWERS, AND TAKING
OUT ALL NAMES AND REFERENCES TO,
AS WE'VE SHOWN IN OUR EARLIER
WORK, BLAH, BLAH, BLAH, SO THAT
ACTUALLY IS TRULY, TRULY BLIND,
BECAUSE I THINK THAT'S THE ONLY
WAY WE'RE ACTUALLY GOING TO GET
EQUITY IN TERMS OF EXCELLENCE.
AND PLAN.
>>TO DO A BLIND REVIEW IN AN
EXPERIMENT, THEY DID -- LOUISE
IS SHAKING HER HEAD.
>>I DID THAT BLIND REVIEW AS A
PILOT, AND IT WAS INCREDIBLY
DIFFICULT.
SO YOU KNOW, WE WERE GIVEN 10
GRANTS OR WHATEVER AND IT WAS
ALL BLOCKED OUT, BUT IT REALLY
WAS DIFFICULT TO ASSESS THE
FEASIBILITY IF YOU DON'T HAVE
ANY EQUIPMENT, ANY RESOURCES,
ANY REFERENCE, YOU CAN TAKE OUT
THE INSTITUTION, BUT THEY EVEN
BLINDED THE BIO SKETCHES,
INCLUDING THE PAPERS.
>>OF COURSE YOU NEED TO KNOW IF
THEY HAVE THE EQUIPMENT TO DO SO
THAT WOULD BE SOMETHING I
WOULDN'T SUGGEST TO TAKE OUT AND
MAKE BLIND, BUT YEAH, YOU'D NEED
SQUARE FOOTAGE AND YOU NEED --
>>YEP, BUT EVEN THE BIO
SKETCHES WERE BLINDED SO YOU
COULDN'T EVEN REALLY
DETERMINE -- INCLUDING ALL THEIR
PAPERS.
BECAUSE THEY HAD THEIR NAMES IN
THEM.
SO I MEAN, I THINK THERE'S A
SMARTER WAY TO DO IT THAN
BLINDING COMPLETELY BECAUSE THEN
IT JUST MAKES IT VERY HARD TO
JUDGE THE FEASIBILITY AND
PRODUCTIVITY OF THE
INVESTIGATOR, AND THOSE SHOULD
FACTOR IN A LITTLE.
>>MAYBE THOSE CAN FACTOR IN
AFTER THE SCORE OF THE ACTUAL
SCIENCE HAS BEEN DONE.
MAYBE DURING THE DISCUSSION OF
THE -- ANYWAY, IT SEEMS LIKE
THERE ARE WAYS TO DO IT AND
THERE ARE PEOPLE WHO STUDY THIS
FULL TIME.
AND I THINK WE SHOULD GIVE IT
ANOTHER TRY MAYBE.
>>OKAY, LOUISE, CORRECT ME IF
I'M WRONG, BUT THE REPORT I
HEARD, THEY DID THIS BLINDED
REVIEW, AND THEN THEY UNBLINDED
TO SEE WHERE THE PEOPLE CAME
FROM, WHAT THEIR REPUTATION WAS,
AND THEY LOOKED AT IT BY RACE
AND ETHNICITY, AND AFRICAN
AMERICANS WAS NO CHANGE, BLINDED
OR UNBLINDED.
CAUCASIANS WENT UP ABOUT
2 POINTS IN THEIR SCORE, WHEN
THEY WENT FROM UNBLINDED TO
BLINDED.
>>AGAIN, IT DEPENDS ON -- THE
DEVIL IS IN THE DETAILS.
IT DEPENDS ON HOW IT'S DONE.
>>I AGREE, GINA.
WE'VE BEEN TALKING ABOUT THIS AT
THE AHA, AS WALTER HEARD A
COUPLE WEEKS AGO, ABOUT DO WE DO
A BLINDED LIKE LETTER OF INTENT
STAGE.
SO WE'RE GOING TO PILOT THAT
OUT, HAVE A BLINDED LETTER OF
INTENT, AND THEN YOU GET THE
INFORMATION YOU NEED BECAUSE I
MEAN, IT IS IMPORTANT TO
SCORE -- TO SEE IF SOMEBODY IS
10 YEARS INTO THEIR RESEARCH
PROGRAM AND HASN'T PUBLISHED ANY
PAPERS, THAT'S NOT, YOU KNOW,
GREAT.
SO IT WAS JUST BASICALLY A WALL
ON THE BIOSKETCH OF BLACKED OUT
STUFF, AND WALTER NOTES, IT
DIDN'T MAKE ANY DIFFERENCE AT
ALL FOR THE THINGS WE WERE
TRYING TO ACHIEVE.
>>RIGHT.
OKAY.
>>BY THE WAY, CSR HAS DONE THE
EXPERIMENT, EXACTLY THE
EXPERIMENT YOU PROPOSED, GINA,
FACTORING IN THE INVESTIGATOR
AFTER REVIEW.
THAT HAS BEEN DONE.
I'M NOT EXACTLY SURE WHAT THEY
LEARNED.
I KNOW ERNIE FROM OUR REVIEW
BRANCH KNOWS ABOUT THAT.
I DON'T KNOW IF HE KNOWS WHAT
THE RESULT WAS OR IF HE CAN GET
ON, BUT IN ANY CASE.
THIS PROBLEM IS BEING THOUGHT
ABOUT INTENSELY AT CSR AND IN
OUR OWN REVIEW BRANCH.
IT'S A BIG DEAL.
WAS THERE ANOTHER HAND UP?
NO, I GUESS NOT.
>>I HAD TWO MORE THINGS,
THOUGH.
>>OH.
KEEP GOING.
>>OKAY.
ONE WAS ABOUT DATA SHARING.
I THINK IT'S WONDERFUL WE'RE
ABOUT TO DO IT OR ARE ALREADY
DOING IT, AND INCREDIBLY
IMPORTANT.
I JUST WONDER IF WE HAVE ENOUGH
RESOURCES DEDICATED TO IT,
BECAUSE IT'S EXPENSIVE TO
CONVERT, YOU KNOW, AND WHAT
HAPPENS TO DATA THAT'S FIVE
YEARS OLD AND THE GRANT FOR IT
HAS NOW ENDED, AND WE'VE
CONVERTED TO A NEW FORMAT FOR
EVERYBODY TO LOOK AT DATA AND
CDs ARE NO LONGER -- NOBODY
HAS A CD PLAYER OR, YOU KNOW,
MAGNETIC TAPES OR WHATEVER.
ALL OF THESE DATA THAT ARE FROM
PAST GRANTS, THEY'RE STILL
EXTREMELY VALUABLE AND
ESPECIALLY AS SCIENCE ADVANCES
AND WE KNOW NOW WHAT TO LOOK FOR
THAT WE DIDN'T KNOW FROM BEFORE,
THE DATA THAT ARE OUT THERE AND
ARE FESTERING AND ROTTING IN
BASEMENTS SOMEWHERE OR BEING
THROWN OUT, IT'S JUST -- IT'S
INCREDIBLY EXPENSIVE TO RE-DO
ALL OF THESE STUDIES INSTEAD OF
JUST, YOU KNOW, PUTTING INTO
OPEN SOURCE ALL OF THIS DATA
THAT ARE ALREADY OUT THERE.
IT KILLS ME TO SEE, YOU KNOW, A
THOUSAND INFANT STUDIES FROM
1970 AND 1980 THROWN AWAY RATHER
THAN JUST MADE AVAILABLE.
SO WHAT ARE WE DOING FOR THAT?
OR WHAT COULD WE DO?
>>OKAY, SO I'M NOT AN EXPERT
HERE, BUT MY UNDERSTANDING WAS
THAT THIS DATA WILL NOW BE ON
OUR WEBSITE SOMEWHERE WITH A
LINK THAT WILL BE IN THE
PUBLICATION.
>>THAT'S WONDERFUL.
THAT'S GREAT.
FROM HERE ON OUT, THAT'S GREAT.
BUT I WAS JUST WONDERING IF WE
CAN HAVE AN INITIATIVE TO SAVE
OLD DATA.
INCLUDING LAB NOTEBOOKS AND ALL
OF THAT.
AND ALSO, I GUESS MAYBE BETTER
GUIDANCE FROM THE NIH ABOUT
WHERE THOSE DATA SHOULD BE
STORED.
IF IT'S ON A COMMERCIAL SITE,
THAT COULD GO DOWN AT ANY
MINUTE, THEN IT'S DONE.
YOU KNOW, MAYBE THE NIH NEEDS TO
INVEST IN ITS OWN DATA FARM
STORAGE OR SOMETHING, SOMETHING
THAT'S LASTING.
JANE'S GOT SOME THINGS TO SAY
ABOUT THAT.
>>YEAH, BUT I'LL JUST SAY ABOUT
DATA SHARING, HAVING SPENT A
GOOD DEAL OF MY TIME ON THAT
TOPIC, THE CHALLENGE WITH
SHARING ALL THE DATA CAN BE
REALLY HARD TO INTERPRET.
IF YOU JUST FIND SOMEBODY'S LAB
NOTEBOOK AND JUST TRY TO
UNDERSTAND WHAT THEIR DATA
MEANT, WHAT CONTEXT IT WAS
COLLECTED, THERE IS SO MUCH
INFORMATION LOST.
SO SOMEHOW AS WE SHARE DATA, YOU
NEED TO KEEP ALL OF THAT
METADATA ALONG WITH IT SO PEOPLE
CAN UNDERSTAND, MAKE SENSE OUT
OF WHAT WAS DONE, BECAUSE
SOMETIMES IT'S WORTH REDOING AN
EXPERIMENT WITH IN TECHNOLOGY,
WE ALL KNOW THIS.
SO THAT'S ONE THING.
THE OTHER THING IS WITH SHARING
DATA, IT'S ALL VERY WELL TO KEEP
IT BUT KEEPING WHAT THEY CALL
FAIR PRINCIPLES, MAKING IT
ACCESSIBLE AND USABLE AT THE
OTHER END, WHICH IS A WHOLE AREA
OF DATA SCIENCE AND ACTUALLY
BEING ABLE TO FIND THAT DATA,
USE THAT DATA AND INTERPRET THAT
DATA.
I CAN GO ON FOR HOURS BUT I
WON'T BECAUSE I'M SURE NOBODY
WANTS THAT BUT IT'S A REALLY
IMPORTANT AREA TO THINK ABOUT,
BECAUSE AS YOU SAY, WE DON'T
WANT TO LOSE THAT INFORMATION
BUT WE DO WANT TO SHARE IT IN A
THOUGHTFUL WAY WHERE PEOPLE CAN
ACTUALLY ACCESS IT AND USE IT.
>>ABSOLUTELY.
THANK YOU SO MUCH, JANE.
I WANTED TO MAKE A PLUG, MAYBE
HANK PAULSON CAN CHIME HERE,
UNIVERSITY OF MICHIGAN HAS
SOMETHING CALLED DEEP BLUE.
IT MIGHT NOT BE THE ONLY ONE,
BUT IT'S GOT A GREAT WAY YOU CAN
REPOSIT YOUR DATA AND YOU FILL
OUT A FORM AND IT'S VERY GENERIC
AND STANDARDIZED SO THAT ANYBODY
CAN ACCESS IT.
HANK, DO YOU WANT TO UNMUTE?
>>I AGREE WITH YOU THAT --
ACTUALLY I THINK THAT
UNIVERSITIES LIKE MICHIGAN,
WHICH HAVE DEEP POCKETS IN THIS
AREA AND EXPERIENCE, I THINK ARE
WELL EQUIPPED TO HELP
INVESTIGATORS PLACE THEIR DATA
IN AN ACADEMIC ENVIRONMENT WHERE
IT'S RELATIVELY CHEAP AND
RELATIVELY ACCESSIBLE.
THAT'S IMPORTANT.
BUT I ALSO SECOND YOUR POINT
ABOUT GINA, NIH AND NINDS MAKING
A COMMITMENT TO THIS, BECAUSE
THIS IS GOING TO BE A NIGHTMARE
IN TERMS OF COST AND
MAINTENANCE.
>>THE HEAD OF OUR DIVISION OF
NEUROSCIENCE, LYN JAKEMAN, HAS
REALLY TAKEN THIS CAUSE ON.
WE HAVE AN INTERNAL WORKING
GROUP LOOKING AT THIS REALLY
INTENSELY.
GOING BACK TO THE LAST THING
WITH BLINDING PEER REVIEW, IF
YOU WANT TO READ THE PAPER I WAS
TALKING ABOUT, IT WAS WRITTEN BY
RICHARD NAKAMORA.
USED TO BE THE HEAD OF CSR.
THE LAST AUTHOR IS BRUCE REID,
NOW DEPUTY DIRECTOR OF CSR.
THE BOTTOM LINE IN THEIR
BLINDING EXPERIMENTS, THAT IT
REDUCED THE REVIEWER'S ABILITY
TO GUESS THE IDENTITY OF THE
P.I. BUT IT DIDN'T ELIMINATE IT.
HOWEVER -- SO I'M GOING TO READ
QUICKLY FROM THE RESULTS --
STANDARD FORMAT APPLICATIONS --
THIS IS THE PREVIOUS, THE USUAL
FORMAT -- FROM WHITE
INVESTIGATORS SCORED BETTER THAN
BLACK INVESTIGATORS WERE
ACTUALLY CUT THE SIZE OF THE
DIFFERENCE BY ABOUT HALF IN
MATCHED APPLICATIONS.
APPLICATIONS FROM WHITE
SCIENTISTS TO SCORE WORSE BUT
HAD NO EFFECT AS WALTER I THINK
MENTIONED OR SCORES FOR BLACK
APPLICANTS.
SO THE PAPER IS IN ELIVE AND THE
FIRST AUTHOR IS RICHARD
NAKAMORA.
IT'S IN THE CHAT, I SEE.
>>THANK YOU, THANK YOU.
AND AGAIN, THAT WAS ONE
EXPERIMENT, MAYBE WE NEED TO DO
A FEW MORE.
>>ABSOLUTELY.
IT'S CRITICAL.
I MEAN, IT TWO THINGS.
FIRST THERE'S BUY A THEN THERE'S
THE ERROR BARS IN PEER REVIEW,
WHICH WE'VE ALSO ATTEMPTED.
TWO DIFFERENT STUDY SECTIONS
REVIEW THE SAME APPLICATION,
WHAT ARE THE ERROR BARS?
ANOTHER CRITICAL POINT.
OKAY.
ANY LAST QUESTIONS ABOUT
WALTER'S TALK?
>>EMAILS, ANY QUESTIONS,
SUGGESTIONS, PLEASE EMAIL ME.
>>OKAY.
WALTER, WE'RE SHIFTING INTO THE
HEAL SEGMENT, AND I THINK YOU
WERE GOING TO INTRODUCE MICHAEL
AND LINDA?
>>JUST TALKING ABOUT STAFF AT
NIH, AND AT NINDS, AND THIS IS
JUST A GREAT EXAMPLE OF HOW OUR
STAFF COMPLETELY REVOLUTIONIZED
AN AREA OF SCIENCE, WHICH IS THE
PAIN SIDE OF SCIENCE.
AND SO YOU'RE GOING TO HEAR FROM
LINDA PORTER, WHO'S THE DIRECTOR
FOR THE NIH OFFICE OF PAIN
POLICY AND PLANNING, AND THE
CO-LEAD FOR THE HELPING END
ADDICTION LONG TERM CLINICAL
PAIN GROUP, AND MICHAEL
OSHINSKY, WHO IS THE HEAD OF OUR
TRANS-NIH PRE-CLINICAL PAIN
RESEARCH AND ALSO THE HEAD OF
THE HEAL TEAM OR THE
PRE-CLINICAL PAIN RESEARCH, AND
WE ARE FUNDING FOR PAIN THROUGH
THE HEAL INITIATIVE, IT'S NOW
$280 MILLION A YEAR, WHICH HAS
ALLOWED THINGS TO BE DONE THAT
YOU COULD NEVER HAVE IMAGINED.
AND IT WAS JUST -- WE WERE
PREPARED.
WE HAD REALLY GOOD PEOPLE, THEY
WERE REALLY KNOWLEDGEABLE, AND
THEN, YOU KNOW, THIS THING FELL
OUT OF THE SKY AND WE WERE ABLE
TO TAKE ADVANTAGE OF IT.
SO EACH YEAR, THEY'LL GIVE A
PRESENTATION, SUMMARY OF WHAT
WENT ON IN THE LAST YEAR.
AND SO LET ME HAND OFF TO LINDA
AND MICHAEL.
>>THANK YOU, WALTER.
CAN WE HAVE THE SLIDES UP?
SO LINDA AND I ARE GOING TO BE
SPEAKING ON THE NIH HEAL
INITIATIVE AND SOME OF THE
ACCOMPLISHMENTS WE'VE HAD IN THE
LAST FISCAL YEAR.
AND WE THANK THE NINDS COUNCIL
FOR THE OPPORTUNITY TO SHOW YOU
SOME OF THESE ACCOMPLISHMENTS
AND TO GET YOUR FEEDBACK ON
THESE PROGRAMS AND SUGGESTIONS
FOR HOW TO MAKE THEM BETTER AND
HOW TO FILL SOME OF THE GAPS
THAT STILL EXIST.
SOG THE NIH HEAL INITIATIVE,
HELPING TO END ADDICTION LONG
TERM'S GOAL IS TO REALLY USE
SCIENCE AND DATA TO ADDRESS THE
OPIOID EPIDEMIC.
BUT NOT JUST THE OPIOID
EPIDEMIC, BUT ALSO THE EPIDEMIC
OF PAIN.
IN ORDER TO ADDRESS THOSE TWO
DIFFERENT ASPECTS OF THESE
EPIDEMICS, MEANING OPIOID
OVERUSE DISORDER AND OF THE PAIN
EPIDEMIC, WE'VE BROKEN UP INTO
DIFFERENT TEAMS.
I'M GOING TO BE PRESENTING THE
PRE-CLINICAL AND TRANSLATIONAL
TEAMS' EFFORT TO ENHANCE PAIN
MANAGEMENT.
YOU CAN SEE HERE ON THE SLIDE
SEVERAL OF THE GOALS THAT WE USE
AS GUIDING PRINCIPLES WHEN WE
DEVELOP OUR FUNDING INITIATIVES
AND MAKE FUNDING DECISIONS.
NOW THESE GOALS CAME FROM A
SERIES OF MEETINGS THAT HAPPENED
IN THE SUMMER OF 2017.
WHERE WE SPOKE TO STAKEHOLDERS,
CLINICIANS, SCIENTISTS, AND
COMPANIES INVOLVED IN THIS
SPACE, MEANING OUD AND
THERAPEUTICS DEVELOPMENT, AND
ASKED THEM WHERE ARE THE GAPS,
WHAT'S NEEDED IN ORDER TO FILL
THOSE GAPS, AND REALLY TO
ACCELERATE GETTING NEW
TREATMENTS INTO THE HANDS OF
PHYSICIANS AND PATIENTS SO THAT
WE CAN CHANGE THEIR LIVES AND
ADDRESS THE SEVERITY OF THIS
EPIDEMIC.
SO I'M GOING TO FOCUS ON THE
PROGRAMS THAT ARE FOCUSED ON
UNDERSTANDING THE BIOLOGICAL
UNDERPINNINGS OF CHRONIC PAIN
AND MORE IMPORTANTLY,
ACCELERATING DISCOVERY AND
PRE-CLINICAL DEVELOPMENT OF
THESE PAIN TREATMENTS THAT HAVE
LITTLE OR NO ADDICTION
LIABILITY.
NEXT SLIDE.
SO THIS CHEVRON DIAGRAM, IF YOU
LOOK AT THE TOP, GOING FROM
DISCOVERY THROUGH PRE-CLINICAL
DEVELOPMENT AND THEN THE INITIAL
CLINICAL TRIALS AND THEN, OF
COURSE, DISSEMINATION OF THAT
RESEARCH IS REALLY A SUMMARY OF
THE DIFFERENT CATEGORIES THAT
THESE FUNDING ANNOUNCEMENTS OR
THESE INITIATIVES CAN FALL INTO.
THE ONES THAT ARE IN M MAGENTA,
THESE 12 THAT ARE LISTED HERE,
REPRESENT THE ONES THAT ARE IN
THE PRE-CLINICAL AND
TRANSLATIONAL PAIN WORK GROUP.
AND THEY SPAN THE RANGE OF
THERAPEUTICS DEVELOPMENT FROM
DISCOVERY THROUGH THE INITIAL
CLINICAL TRIALS.
AND THEN LINDA WILL BE
DISCUSSING THE OTHER PROGRAMS
THAT ARE LISTED HERE IN GREY.
NOW, IF YOU START FROM THE TOP,
YOU CAN SEE THAT THERE'S A
PROGRESSION FROM TARGET
DISCOVERY AND VALIDATION OF
THOSE TARGETS THROUGH INITIAL
ANALGESIC DEVELOPMENT, AND THEN
A PAIN THERAPEUTICS DEVELOPMENT
PROGRAM WHICH CAN BRING A TEAM
TO THEIR PHASE ONE AND THEIR
INITIAL CLINICAL TRIALS.
NOW, EVEN THOUGH THE PROGRAMS
ARE DESIGNED FOR TAKING A
PROGRAM ALONG THIS PATH, THEY'RE
ALSO DESIGNED TO TAKE IN ASSETS
OR IDEAS AT ANY PARTICULAR
STAGE.
SO THERE'S NO REQUIREMENT THEY
GO IN IN THIS PARTICULAR ORDER.
WE ALSO HAVE PROGRAMS SUCH AS
THE PRE-CLINICAL SCREENING
PLATFORM FOR PAIN, THE PSPP
PROGRAM, WHERE WE PROVIDE A
PROFILING PLATFORM FOR
INDIVIDUALS WHO MIGHT HAVE A
LATER STAGE ASSET AND HELPING
THEM TO MAKE A DECISION ABOUT
WHETHER OR NOT IT WOULD BE
HELPFUL OR POTENTIALLY
SUCCESSFUL TO INVESTIGATE THIS
PARTICULAR ASSET IN A PAIN
THERAPEUTIC CONDITION.
ON THE LOWER LEFT-HAND PART OF
THE SLIDE, YOU SEE THE PRECISION
HUMAN PAIN INITIATIVE, THE
REJOIN, THE ANIMAL MODEL
DEVELOPMENT.
THESE ARE ALL SPECIFIC PROGRAMS
WHICH WE SEE AS FEEDING IN DATA
OR INFORMATION, OR IN THE CASE
OF AN MOL M ANIMAL MODEL DEVELO,
VALIDATED MODELS IN ORDER TO
ENHANCE THE PROGRAMS THAT ARE IN
THOSE INITIATIVES TO MAKE THEM
MORE SUCCESSFUL AND HOPEFULLY
GIVE US AN IDEA AND HELP US
PREDICT WHETHER OR NOT THEY'LL
BE SUCCESSFUL IN PATIENTS.
SO I'M GOING TO DISCUSS
INITIALLY THE PAIN TARGET
DISCOVERY AND VALIDATION
PROGRAMS.
THERE'S WHO PARTS YOU SEE THEM
BROKEN INTO.
ON LEFT SIDE YOU SEE THE
DISCOVERY AND VALIDATION OF
NOVEL TARGETS.
THE FUNDING ANNOUNCEMENTS HAVE
BEEN ISSUED OVER THE LAST FOUR
YEARS, IN THE FORM OF
SUPPLEMENTS INITIALLY, R01s
AND R21s TO REALLY VALIDATE
TARGETS AND TO PRODUCE A DATASET
IN ORDER TO HELP THOSE WHO WERE
DOING THERAPEUTICS DEVELOPMENT
DETERMINE WHETHER OR NOT IT'S
WORTH BUILDING AN ASSET OR
DEVELOPING TOOL COMPOUNDS FOR
THIS ASSET IN ORDER TO SEE
WHETHER OR NOT THEY'LL WORK IN
ANIMAL MODELS OR IN THANK YOU
MAN THUMANTISSUES OR THE VALIDAE
MODELS WE HAVE.
THE GOAL OF THIS IS TO PRODUCE
DATASETS TO CONVINCE OTHERS TO
DO THERAPEUTIC DEVELOPMENT.
WE HAVE DONE 56 AWARDS IN THIS
AREA, WHICH REPRESENT 25
DIFFERENT TYPES OF PAIN AND 55
DIFFERENT TARGETS.
SO THERE'S MANY TARGETS THAT ARE
IN THE PROCESS OF BEING
VALIDATED HERE.
AND WHAT WE'RE DOING ON THE
FRONT END IS TRYING TO PRODUCE A
HUGE DATASET OF VALIDATION FOR
THESE MANY TYPES OF TARGETS, AND
MANY PAIN CONDITIONS, IN ORDER
TO MAKE SURE THAT THERE ISN'T
JUST A PIGEONHOLING OF THE,
LET'S SAY, THE MOST POPULAR PAIN
CONDITIONS OR THE ONES THAT WE
THINK ARE THE MOST IMPORTANT,
BUT REALLY HAVING THE SCIENCE OF
WHETHER OR NOT THINGS ARE
ACTUALLY CAPABLE OF MAKING IT
THROUGH THE CLINICAL TRIALS AND
VALIDATED IN ORDER TO DRIVE
WHETHER OR NOT WE SUPPORT THE
PROJECT OR WHETHER OR NOT IT
SHOULD GO INTO THERAPEUTICS
DEVELOPMENT.
AN IMPORTANT PART OF THOSE
R01s ALSO IS THE TYPE OF
VALIDATION.
SO AS A PART OF THE HEAL
INITIATIVE IN 2020, WE CONVENED
A WORKSHOP WITH INDUSTRY MEMBERS
AND ACADEMICS AND BROUGHT THEM
TOGETHER AND ASKED THEM THE
QUESTION, WHAT TYPE OF
VALIDATION IS REALLY NEEDED TO
HELP WITH THAT PREDICTION ABOUT
WHETHER OR NOT THE PHASE TWO OR
THE LATER CLINICAL TRIALS ARE
GOING TO BE SUCCESSFUL.
AND WE RECENTLY HAD A
PUBLICATION IN THAT AREA, AND
THAT WAS LED BY -- OR DIRECTLY
LED BY INDUSTRY MEMBERS AND THE
DIVISION FOR TRANSLATIONAL
RESEARCH TEAM, AMIR TAMIZ AND
HIS GROUP.
SO WE'RE REALLY PROUD OF THAT
PUBLICATION, IT'S A GUIDE FOR
THE COMMUNITY AND IT ALSO GUIDED
THESE PARTICULAR FUNDING
ANNOUNCEMENTS.
ON THE RIGHT-HAND SIDE OF THE
SLIDE, THIS PRECISION HUMAN PAIN
NETWORK, PROGRAM TO REVEAL AND
EVALUATE CELLS TO GENE
INFORMATION THAT SPECIFY
INTRICACIES AND ORIGINS AND THE
NATURE OF HUMAN PAIN.
IT'S A MOUTHFUL, BUT IT REALLY
SAYS A LOT AND THAT'S WHY I
SPELLED IT OUT.
THIS INITIATIVE IS FUNDING
GROUPS THAT ARE TAKING
EXCLUSIVELY HUMAN TISSUE FROM
INDIVIDUALS WITH PATHOLOGICAL
PAIN CONDITIONS, TRIGEMINAL PAIN
CONDITIONS, SOMATIC PAIN
CONDITIONS, OSTEOARTHRITIS, LOW
BACK PAIN, HEADACHE, TMD, ET
CETERA, ET CETERA.
AND REALLY USING THE TISSUE FROM
THOUSANDS OF PATIENTS, AND DOING
CELLULAR FUNCTIONAL PHENOTYPES,
MOLECULAR SIGNATURES, PROTEOMICS
AND EVEN IN SOME I CASES
TRANSCRIPTOMICS, AND PRODUCING
DATASETS.
THAT'S THE GOAL OF THESE
PROGRAMS, IS TO PRODUCE DATASETS
THAT WILL BE SHARED WITH OTHERS
IN ORDER TO HELP THEM DEVELOP
THE VALIDATION OR TO VALIDATE
THE TARGETS THAT THEY MIGHT HAVE
FOUND IN AN ANIMAL MODEL IN
ORDER TO HELP THEM WITH THE
THERAPEUTICS DEVELOPMENT
PROCESS.
WE ALSO HAVE A GOAL OF PRODUCING
THIS DATA COORDINATING CENTER TO
HELP BUILDING KNOWLEDGE BASES,
HARMONIZATION OF THE DATA, AND
ENHANCE THE SHARING OF IT AND
CREATE THESE DIGITAL RESOURCES
FOR THE INTEGRATION,
VISUALIZATION AND DISSEMINATION
OF THIS WORK TO INSPIRE OTHERS
TO COME INTO THE PAIN FIELD THAT
MAYBE DON'T HAVE INITIALLY THE
PAIN EXPERTISE BUT HAVE THE
COMPUTATIONAL EXPERTISE, ET
CETERA, THAT HELP US DISCOVER
WHICH ARE THE BEST TARGETS FOR
THESE PAIN CONDITIONS.
THIS SLIDE SHOWS SOME OF THE
2023 INITIATIVES WE HAVE TO
FACILITATE THIS DISCOVERY AT THE
EARLY STAGE.
ON THE LEFT SIDE, WE HAVE TWO
DIFFERENT TYPES OF INITIATIVES
TO SUPPORT DEVELOPING ANIMAL
MODELS, IN THIS CASE, NON-RODENT
ANIMAL MODELS.
CURRENTLY THE RODENT MODELS
OVERWHELMINGLY DOMINATE THE
RESEARCH THAT'S DONE IN THE
PRE-CLINICAL PAIN SPACE.
BUT TO REALLY VALIDATE AND
DEVELOP NON-RODENT MODELS THAT
COULD BE USED FOR THERAPEUTICS
DEVELOPMENT, AND THEN WE HAVE A
NOTICE OF SPECIAL INTEREST,
THESE NOSIs, TO DEVELOP AND
EVALUATE PAIN RELATED MODELS AND
END POINTS TO FACILITATE THIS
ANALGESIC DISCOVER REAL FOR
ASSETS THAT HAVE LITTLE OR NO
ADDICTION LIABILITY.
SO THAT'S A PART OF THE IGNITE
PROGRAM, AND THOSE ARE
MILESTONE-DRIVEN, AND THEN WE
ALSO HAVE THE R01 PROGRAM FOR
DEVELOPING THESE NON-RODENT
MAMMALIAN PAIN MODELS.
ON THE RIGHT SIDE OF THE SLIDE,
WE HAVE THE INTEGRATED BASIC AND
CLINICAL TEAM-BASED RESEARCH IN
PAIN.
AND THIS IS AN RM1 MECHANISM.
AND THE POINT HERE IS TO COMBINE
IN ONE SINGLE PROJECT THE
CLINICAL AND BASIC SCIENCE
RESEARCH.
NOT PARALLEL PROJECTS, BUT
REALLY A COMBINED PROJECT.
AND THIS IS TO ESTABLISH THESE
COMPREHENSIVE UNDERSTANDING OF
HUMAN PAIN BIOLOGY, WHICH WE MAY
NEED A MODEL SYSTEM TO ANSWER A
QUESTION THAT WAS IDENTIFIED IN
THE HUMAN CONDITION.
SO YOU HAVE A CLINICIAN AND YOU
HAVE A BASIC SCIENTIST OR A
CLINICIAN-SCIENTIST WITH A BASIC
SCIENTIST TO ANSWER THESE
QUESTIONS IN ONE PARTICULAR
PROJECT.
THE EMPHASIS IS
BACK-TRANSLATION.
THIS IS ANOTHER RECOMMENDATION
THAT WE HAD FROM THAT WORKSHOP
THAT WE DID IN 2020, THAT THOSE
IN PHARMA, THOSE IN BIG PHARMA
ARE REALLY LOOKING FOR THESE
VALIDATED TARGET, HUMAN TISSUE,
THAT REALLY HAVE A CLINICAL
BASIS FOR THE RATIONALE FOR
WORKING UP THAT TARGET FOR
THERAPEUTICS DEVELOPMENT.
THE NEXT PROGRAM I'M GOING TO
TALK ABOUT IS TRANSITION FROM
THAT BASIC BIOLOGY,
UNDERSTANDING THAT THE
PATHOLOGICAL CONDITION FROM THE
TARGET MOLECULAR LEVEL, ET
CETERA, AND THEN ASKING THE
QUESTIONS, WHAT STEPS ARENESS
SAIR HEARENECESSARY IN ORDER TO,
TO BEGIN, A THERAPEUTICS
DEVELOPMENT PROGRAM.
AND WE HAVE TWO DIFFERENT
GRANTING MECHANISMS IN THIS AREA
TO HELP THE INDIVIDUALS BRIDGE
THAT VALIDATED TARGET TO THEN
PLAN AN INITIAL THERAPEUTICS
DEVELOPMENT PROGRAM AND THEN
HAVE THE TEAM TOGETHER THAT
INCLUDES FORMULATION EXPERTISE,
CHEMISTRY, THE PAIN BIOLOGY,
MAYBE THAT CLINICIAN, AND EVEN A
PATIENT ADVOCATE THAT CAN INFORM
WHAT THE PATIENT REALLY NEED
FROM THE THERAPEUTIC.
SO WE HAVE TWO PROGRAMS TO DO
THAT.
ONE IS A TWO-YEAR PLANNING
GRANT, THE OTHER IS A FIVE-YEAR
MULTICOMPONENT PROGRAM THAT
SUPPORTS THE DEVELOPMENT OF
ASSAYS DOING SCREENING, EARLY
OPTIMIZATION, EVEN
PHARMACODYNAMIC BIOMARKER
DISCOVERY, PK/PD STUDIES, ET
CETERA.
SO THERE'S INITIAL STUDIES TO
GET THE PROGRAM GOING, AND THEN
THOSE PROGRAMS, THE GOAL HERE IS
TO FEED THEM INTO THE PAIN
THERAPEUTICS DEVELOPMENT
PROGRAM, THE PTDP PROGRAM.
ON THE PLANNING GRANTS -- THIS
IS REALLY TEAM SCIENCE.
SO THE PAIN THERAPEUTICS
DEVELOPMENT PROGRAM IS REALLY
BASED ON THE BLUEPRINT
NEUROTHERAPEUTICS PROGRAM WHICH
YOU'VE HEARD ABOUT FOR MANY
YEARS AT NINDS AND ACROSS THE
NIH INSTITUTES.
AND WE SPECIALIZED THIS PROGRAM
FOR DEVELOPING THERAPEUTICS,
SMALL MOLECULES AND BIOLOGICS
FOR TREATING PAIN.
NOW, THIS USES THE UG3,
UH3 PHASED MECHANISM AND JUST
LIKE IN THE BPM PROGRAM, IT'S A
CONSULTANT GROUP TO HELP
INDIVIDUALS WITH THEIR
THERAPEUTICS DEVELOPMENT.
THERE'S CONTRACTS THAT WE USE,
AND MOST OF THE WORK IS DONE
WITH THE CONTRACTS, THEY HAVE
OPPORTUNITY TO DO THEIR
CHEMISTRY THERE, THEIR TOX
STUDIES, ET CETERA, AND THIS
WILL BRING THEM FROM LEAD
OPTIMIZATION, LET'S SAY THEY
COME IN IN THE DISCOVERY STAGE,
IND ENABLING STUDY, WE CAN DO
THAT ALL WITH OUR CONTRACTS, AND
PROBABLY MOST IMPORTANTLY IS IF
THEY GET TO THE STAGE, WHERE
THEY CAN DO THAT SUBMISSION FOR
THEIR IND IS WE ALSO HAVE A
CONTRACT TO HELP THEM WITH THEIR
PHASE ONE CLINICAL TRIALS.
SO IT REALLY A SOUP TO NUTS
SERVICE FOR THE GROUPS WITH
THERAPEUTICS DEVELOPMENT
EXPERTISE OR IF THEY ONLY HAVE A
LITTLE BIT OF THAT EXPERTISE,
THAT THEY CAN GET THAT
CONSULTANT AND EXPERTISE FROM
THE NIH STAFF, WHICH THE STAFF
THAT RUNS THIS HAS LOTS OF FARM
MA EXPERIENCE, AND PAIN PHARMA
EXPERIENCE, BRING THAT TO THEIR
PROJECTS AND REALLY BRING THAT
EXPERIENCE.
YOU CAN SEE THE MANY PAIN
CONDITIONS WE HAVE REPRESENTED
HERE, AND EVEN THOUGH MANY OF
THESE PAIN CONDITIONS ARE NOT IN
NINDS'S MISSION, IT'S NINDS WITH
THE OTHER INSTITUTES THAT DO
HAVE THESE PAIN CONDITIONS IN
THEIR MISSION THROUGH THE HEAL
INITIATIVE, WE CAN BRING THOSE
GROUPS TOGETHER TO GET TO THIS
GOAL OF GETTING NEW TREATMENT,
YOU KNOW, VALIDATED AND IN THE
HANDS OF THE PHYSICIANS.
SO I STATED THAT THE GOAL WAS TO
REALLY GET THE TREATMENTS INTO
THE HANDS OF THE PHYSICIANS AND
PATIENTS.
WE CANNOT DO THAT WITHOUT
COMMERCIAL BUSINESSES.
AND THE SBIR PROGRAM IN THE HEAL
INITIATIVE HAS REALLY BEEN
TREMENDOUSLY SUCCESSFUL.
WE'VE HAD 172 COMPANIES THAT
HAVE APPLIED FOR THESE PROGRAMS.
WE FUNDED 46 OF THESE COMPANIES
FOR 49 DIFFERENT PROJECTS.
SPANNING THE PAIN INTEREST
ACROSS NINE DIFFERENT NIH
INSTITUTES, AND THAT'S SOMETHING
THAT'S REALLY SPECIAL ABOUT THE
NIH HEAL INITIATIVE, THAT IT'S
TRANS-NIH, AND THAT WE CAN TAKE
AWAY THE DISCUSSION OF THE
MISSION RELEVANCE WHEN IT COMES
TO WHAT TYPE OF PAIN, AND JUST
REALLY ASK THE QUESTION, WHAT'S
THE POTENTIAL OF THIS PARTICULAR
PROJECT TO MAKE A DIFFERENCE FOR
THOSE WHO ARE SUFFERING FROM
PAIN.
AND OF COURSE THE OPIOID
EPIDEMIC.
WE'VE ALLOCATED THROUGH THE SBI
PROGRAM $38 MILLION AT THIS
POINT AND IT ACTUALLY EXCEEDS
THAT, THE HEAL OD AND THE
EXECUTIVE COMMITTEE HAS REALLY
SEEN THE POTENTIAL FOR THESE
PROGRAMS AND THERE'S BEEN A
GREATER ALLOCATION THAN THAT
REQUIRED MINIMUM, LET'S SAY.
LET'S GET N INTO MORE OF THE
DETAILS WE'VE HAD IN THE SBIR.
ONE OF THE REAL BENCHMARKS OF
THE SUCCESS OF THESE
THERAPEUTICS DEVELOPMENT
PROGRAMS IS GETTING THAT
TRANSITION FROM THE SCIENTIFIC
QUESTION AND DEVELOPMENT OF AN
ASSET AND ASKING IS IT READY FOR
THE CLINIC.
THAT'S WHAT BIG PHARMA IS
TELLING US THEY WANT TO SEE.
THEY WANT TO SEE THE HUMAN DATA,
THEY WANT TO SEE THE EFFECT IN A
THERAPEUTIC PAIN CONDITION.
SO WE HAVE THE AFASCI COMPANY
THAT HAS DEVELOPED A CAV3
T-CHANNEL MODULATOR FOR THE
TREATMENT OF NEUROPATHIC PAIN,
AND THEY'VE RECEIVED THEIR IND
SO IT'S IN ACTIVE STATUS WITH
THE FDA, AND THE COMPANY IS
CURRENTLY SEEKING FUNDING FOR
THAT PHASE ONE CLINICAL TRIAL.
WE ALSO HAVE SOUTH RAMPART
PHARMA, WHICH HAS AN ASSET, THIS
SRP-3D, IT'S NOT ITS IND
APPROVAL, AND THEY'RE CURRENTLY
IN RECRUITMENT FOR THEIR PHASE
ONE CLINICAL TRIAL, AND THIS HAS
ALL BEEN BROUGHT TO THIS STAGE
WITH HEAL SBIR MONEYS, AND WE
ALSO HAVE MY CO-LEADS, WHICH IS,
WHICH IS A DEVICE PROGRAM WHICH
HAS THEIR IDE APPROVAL, AND HAS
INITIATED THE NEXT PHASE OF
CLINICAL TESTING TO INVESTIGATE
FEASIBILITY AND EFFICACY.
THE LAST THING I HAVE ON THE
SLIDE IS COMMERCIALIZATION OF A
PLATFORM OF HUMAN ON A CHIP TO
BE AN AID FOR DRUG DISCOVERY.
SO WE KNOW THAT THE FDA HAS PUT
OUT RECENT GUIDANCE THAT THEY NO
LONGER REQUIRE ANIMAL TESTING
BEFORE YOU GO TO HUMAN DRUG
TRIALS.
AND USING THE SBIR HEAL MONEY,
WE'VE CONTRIBUTED TO ONE OF
THESE PLATFORMS THAT COMPANIES
CAN USE TO HELP THEM MAKE THEIR
GO/NO-GO DECISIONS USING THIS
SILICA PLATFORM THAT WAS
DEVELOPED ALSO IN PARTNERSHIP
WITH NCATS.
SO AFTER THAT STAGE, AFTER YOU
GET TO CLINICAL TESTING, ET
CETERA, YOU STILL HAVE TO
COMMERCIALIZE THE PRODUCT.
AND IF IT'S NOT COMMERCIALIZED,
IT'S STILL NOT GOING TO GET INTO
THE HANDS OF THE PATIENTS.
SO THIS GRAPHIC ON THE RIGHT
SIDE IS REALLY INTERESTING.
YOU SEE THERE IN THE RED, THOSE
INDIVIDUALS ARE THE SCIENTISTS.
AND THESE SMALL COMPANIES HAVE
LOTS OF HUMAN CAPITAL WHEN IT
COMES TO SCIENTIFIC EXPERTISE.
BUT LITTLE OF THAT
COMMERCIALIZATION CAPITAL OR THE
EXPERIENCE NEEDED TO BRING
THINGS TO THE MARKET, AND WE'VE
DEVELOPED THESE TWO PROGRAMS
HERE, IF YOU LOOK AT THE ONE ON
THE BOTTOM, THIS EMBEDDED
ENTREPRENEUR PROGRAM FOR THESE
COMPANIES TO APPLY FOR FUNDING,
TO BRING IN AN EXPERT
ENTREPRENEUR INTO THEIR GROUP TO
CAPITALIZE ON THE FEDERAL FUNDS
THAT WERE THERE, WHO WILL KNOW
HOW TO GET PRIVATE FUNDING,
WHICH CAN THEN BRING THEM TO A
STAGE OF COMMERCIALIZATION.
AND THEN WE'RE ALSO PLANNING IN
OCTOBER 2023 THE HEAL PAIN
PARTNERING COMMITTEE, WHERE WE
CAN SHOWCASE HEAL FUNDED
TECHNOLOGIES, PUT THEM IN FRONT
OF PHARMA COMPANIES TO
FACILITATE PARTNERING, AND OF
COURSE EXPLORE OPPORTUNITIES TO
HEAR FROM THESE COMPANIES AND --
LARGE AND SMALL, WHAT THE
BARRIERS ARE TO MAKING THE TRAN
CIRCLES AND THEN HOPEFULLY
SUPPLEMENT THESE COMPANIES TO
HELP THEM IN SOME WAY TO DO THAT
AND PROVIDE, YOU KNOW,
ADDITIONAL INITIATIVES TO BRING
THEM TO THE NEXT STAGE.
AND THESE PROGRAMS, AGAIN, ARE
IN PARTNERSHIP WITH NCATS AND
NINDS.
NINDS AND THE HEAL INITIATIVE
DOESN'T JUST PROVIDE FUNDS.
THE HEAL INITIATIVE HAS ALSO
PROVIDED US WITH THE ABILITY TO
DEVELOP THE PRE-CLINICAL
SCREENING PLATFORM FOR PAIN,
WHICH IS A PROFILING PROGRAM FOR
INDIVIDUALS WHO HAVE ASSETS
ALREADY.
MAYBE BEING DEVELOPED FOR
ANOTHER NEUROLOGICAL CONDITION.
AND THEY WANT TO ASK, CAN IT
BE -- IS IT WORTH PUTTING THE
EFFORT INTO -- OR THE FUNDS TO
DEVELOP THIS ASSET FOR A PAIN
CONDITION, FOR A PATHOLOGICAL
PAIN CONDITION.
SO THIS PROGRAM CAN PROVIDE
PHARMACOKINETIC STUDIES, IN
VITRO SCREENING, TESTING IN
ANIMAL MODELS, WHICH WE HAVE A
CONTRACTED SITE THAT'S DONE A
REAL EXCELLENT JOB IN VALIDATING
THEIR MODELS AND BEING
CONSISTENT WITH THEM, CONSTANTLY
RUNNING POSITIVE AND NEGATIVE
CONTROLS, ET CETERA, ET CETERA,
SO WE'RE VERY CLEAR ON THE DATA
AND HOW IT COMES OUT IN THE MANY
MODELS.
SOME OF THEM ARE ACUTE MODELS,
OTHERS ARE CHRONIC MODELS, AND
WE HAVE THIS TIERED SYSTEM THEY
CAN GO THROUGH IN ORDER TO
DETERMINE WHETHER OR NOT THERE'S
A PARTICULAR TYPE OF PAIN, IS IT
INFLAMMATORY PAIN OR CHRONIC
PAIN, WE CAN GIVE THEM THIS
DATASET AND DE-RISKING THEIR
DECISION-MAKING OF WHETHER OR
NOT THEY WANT TO PURSUE THIS
ASSET IN PAIN.
UP TO THIS POINT, WE'VE BEEN
TALKING ABOUT VALIDATING
TARGETS, SMALL MOLECULES AND
BIOLOGICS.
THIS PARTICULAR SLIDE SUMMARIZES
THE DEVICE PROGRAMS FOR
DEVELOPING TREATMENTS FOR PAIN
INSIDE THE HEAL INITIATIVE.
AND WE TAKE ADVANTAGE OF
NIH-WIDE PROGRAMS SUCH AS THE
BLUEPRINT MED TECH PROGRAM.
WE ALSO HAVE HAD AN R-18 PROGRAM
FOR DEVELOPING EARLY STAGE
TECHNOLOGIES TO ADVANCE DEVICES
FOR TREATING PAIN.
THERE'S A LONG HISTORY OF
DEVICES FOR THE TREATMENT OF
PAIN.
SPINAL CORD STIMULATORS HAVE
BEEN AROUND FOR A LONG TIME.
TENS UNITS, I'M SURE YOU'VE
HEARD OF THEM IN THE PAST, FOR
STIMULATING PERIPHERAL NERVES
FOR TREATING PAIN.
IN SOME CASES, NON-IN NONINVASI.
THERE'S VAGUS NERVE STIMULATORS
ON THE EAR, THE NECK, THE
PERIPHERAL NERVE IN THE ARM --
ET CETERA.
WE'VE EVEN DEVELOPED A PROGRAM
TO UNDERSTAND THE MECHANISM OF
THESE FDA-CLEARED DEVICES IN
ORDER TO ALLOW US TO DEVELOP
MORE EFFICACIOUS ONES AND BETTER
IDENTIFY WHICH PATIENTS CAN
BENEFIT FROM THEM.
SO WE HAVE SIX DEVELOPMENT
PROJECTS IN THE DEVICE SPACE
CURRENTLY ONGOING.
EIGHT CLINICAL TRANSLATION
PROJECTS.
THE AND SIX EARLY FEASIBILITY
CLINICAL TRIALS.
IN ADDITION TO THAT, WE'VE HAD
FIVE FDA IDEs DEVELOPED
THROUGH THE PROGRAM, TWO ONGOING
MECHANISTIC TRIALS, AND THREE
AWARDS THROUGH THAT SMALL --
PROGRAM AND 14 OF THEM IN
ACADEMIA.
SO WE'RE REALLY EXCITED ABOUT
THIS PROGRAM, YOU CAN SEE ON THE
RIGHT SIDE ON THE BOTTOM THERE
SOME OF THE DIFFERENT PAIN
SYNDROMES THAT ARE INCLUDED, NOT
JUST THE STANDARD LOW BACK PAIN
BUT LIKE I MENTIONED ALSO, SOME
OF THE UNDER D- UNDERSTUDIED PAN
CONDITIONS, IRRITABLE BOWEL
SYNDROME, NEUROPATHIC PAIN, VERY
IMPORTANT, AND REFRACK TRILL
CHRONIC PAIN, DEEP BRAIN
STIMULATION.
THIS SLIDE IS JUST TO SUMMARIZE
THE DISTRIBUTION OF ACADEMIA AND
INDUSTRY THAT WE SEE PURSUING
THE DIFFERENT INITIATIVES THAT
WE'VE PUT OUT.
AS YOU CAN IMAGINE, DISCOVERY
AND VALIDATION IS ALL ACADEMIA.
BUT WE HAVE ABOUT A 70/30 SPLIT
WHEN IT COMES TO THERAPEUTIC
DEVELOPMENT FOR SMALL KNOLL MOLS
AND BIOLOGICS, AND THE PSP
PROGRAM -- BY DEFINITION,
THEY'VE BEEN ALL COMPANIES.
SO HEAL IS REALLY LOOKING AT A
COMPREHENSIVE WAY TO MAKE SURE
THAT THESE DISCOVERIES WE'RE
MAKING AT THE EARLY STAGE HAVE
THE POTENTIAL TO MAKE TO
COMMERCIALIZATION.
I'M GOING TO SWITCH GEARS AS I
TRANSITION TO HANDING IT OVER TO
LINDA PORTER, AND THAT IS TO
DISCUSS WORKFORCE DEVELOPMENT.
WE KNOW THAT IN ORDER TO GET NEW
IDEAS INTO THIS SPACE, TO GET
CREATIVE IDEAS, WE HAVE TO
SUPPORT A DIVERSE WORKFORCE.
AND AT THIS POINT, ONE OF THE
WAYS WE DO THAT IN THE HEAL
INITIATIVE IS THROUGH
SUPPLEMENTS TO THE ONGOING
PROJECTS.
AT THIS POINT, SINCE 2019, WHEN
THE PROGRAM WAS LAUNCHED, WE
SUPPORTED 30 DIVERSE INDIVIDUALS
TO THESE PROGRAMS, AND THAT'S
SOMETHING WE'RE REALLY PROUD.
THE VAST MAJORITIES ARE -- WELL,
ABOUT HALF OF THEM ARE PH.D.
STUDENTS, AND THE OTHER HALF ARE
EARLY STAGE INVESTIGATORS, MANY
OF THEM IN EARLY STAGE OF THEIR
ACADEMIC APPOINTMENT.
ANOTHER WAY THAT WE SUPPORT THE
WORKFORCE IS THROUGH THE K-99R0H
DR. KOROSHETZ MENTIONED IN HIS
INTRO REMARKS.
THIS IS REALLY IMPORTANT FOR
GETTING PEOPLE TO TRANSITION
INTO THE FIELD AND TO STAY INTO
THE FIELD.
WE HAVE A STANDARD AND A
DIVERSITY VAR YAITIO VARIATION O
TAKE INDEPENDENT BASIC SCIENCE
STUDIES OR CLINICAL STUDIES OR
THOSE WHO WANT TO DO THOSE TYPE
OF RESEARCH AND SUPPORT THEM
FROM THERE FROM THEIR MENTORED
STAGE INTO THE UNMENTORED STAGE
OF THEIR CAREER.
SO THIS IS PROBABLY THE MOST
IMPORTANT SLIDE.
ON THE LEFT SIDE WE HAVE THE
INITIATIVE LEADS, AND THEN WE
HAVE THE TEAM MEMBERS ON THE
RIGHT, AND I HAVE TO TELL YOU,
NOT EVERYBODY IS MENTIONED HERE.
OF COURSE IT INCLUDES THE VAST
MAJORITY OF THE PROGRAM PEOPLE,
BUT IT DOESN'T INCLUDE THE
REVIEW BRANCH, WHICH PLAYS A
TREMENDOUS ROLE HERE, AS WELL AS
GMB, AND SENIOR LEADERSHIP AND
ACROSS MANY, MANY DIFFERENT
INSTITUTES YOU SEE LISTED HERE.
BUT THAT MIDDLE COLUMN, THE LAST
TWO YOU SEE THERE, DR. ELIZABETH
SYPEK AND DR. RACHEL WEINBERG
ARE THE HPSs IN THE
PRE-CLINICAL PAIN OFFICE THAT
ARE REALLY THE GLUE FOR ALL OF
THESE PROGRAMS AND MAKE THEM
SUCCESSFUL.
SO THANKS FOR EVERYBODY ON THE
TEAM AND THANKS FOR THE
OPPORTUNITY TO EXPLAIN THESE
PROGRAMS TO YOU.
>>LINDA, CLINICAL RESEARCH.
>>THANK YOU FOR SUCH A NICE
LEAD-IN TO THE CLINICAL
COMPONENTS.
COULD WE GO TO THE NEXT SLIDE?
BEFORE I START, I WOULD LIKE TO
PAY RECOGNITION TO RON DUBNER,
WHO WAS A REALLY VALUABLE,
INNOVATIVE PIONEER IN PAIN
RESEARCH.
HE PASSED AWAY LAST WEEK.
AND SO TO MENTION HIS
ACCOMPLISHMENTS AND
CONTRIBUTIONS I THINK WOULD TAKE
AN HOUR, BUT I DID WANT TO
MENTION QUICKLY THAT HE STARTED
HIS CAREER AT NIH AND SPENT MOST
OF HIS CAREER HERE, STARTED AS A
STAFF DENTIST IN THE CLINICAL
CENTER, AND MOVED ALL THE WAY UP
TO LEADING WHAT TURNED OUT TO BE
A REALLY, REALLY INFLUENTIAL
PAIN RESEARCH PROGRAM IN THE
INTRAMURAL SIDE OF THE NATIONAL
INSTITUTE ON DENTAL RESEARCH.
SO RON'S CONTRIBUTIONS PROBABLY
HAVE MADE PART OF THE BASIS FOR
WHAT WE KNOW NOW TO HAVE BEEN
ABLE TO MOVE THE SCIENCE
FORWARD.
SO WITH THAT, OUR TIME IS
LIMITED SO IF I CAN GO TO THE
NEXT SLIDE, PLEASE.
AS SORT OF A PREVIEW WHY THIS IS
SO IMPORTANT FROM THE PUBLIC
HEALTH SIDE, I HAVE A LITTLE BIT
OF DATA THAT I WANT TO SHARE AND
GIVE YOU A SENSE OF JUST HOW BIG
THE PUBLIC HEALTH CRISIS THAT
MICHAEL MENTIONED REALLY IS.
SO WE KNOW THAT 50 MILLION
ADULTS LIVE WITH CHRONIC PAIN,
DEFINED AS PAIN THAT WAS MOST OR
ALL DAYS IN THE LAST THREE
MONTHS.
HALF OF THOSE HAVE SEVERE PAIN
EVERY DAY AND A LARGE PORTION OF
THEM HAVE HIGH IMPACT CHRONIC
PAIN, DEFINED AS PAIN THAT
AFFECTS THEIR LIFE.
INCLUDING ACTIVITIES OF DAILY
LIVING, SOCIAL ENGAGEMENT AND
WHETHER OR NOT THEY CAN GO TO
WORK, SCHOOL, ET CETERA.
MORE WOMEN THAN MEN, SLIGHTLY
HIGHER PREVALENCE, HAVE CHRONIC
AND -- IMPACT CHRONIC PAIN AND
RURAL MORE THAN URBAN DWELLERS
REPORT CHRONIC PAIN AND HIGH
IMPACT CHRONIC PAIN, PROBABLY
BECAUSE OF ACCESS DIFFICULTY TO
REALLY QUALITY HEALTHCARE.
IF YOU LOOK AT THE UPPER
RIGHT-HAND SIDE, WE KNOW THAT
PEOPLE WHO HAVE OTHER PAIN --
OTHER CHRONIC CONDITIONS THAT
REALLY ARE SEVERE, OFTEN ARE
ALSO HAVING CHRONIC PAIN OR HIGH
IMPACT CHRONIC PAIN ASSOCIATED
WITH THAT.
THIS IS AN ODDS RATIO CHART, AND
FOR SOME OF THOSE CONDITIONS AND
SOME OF THE PEOPLE WHO HAVE
THOSE CONDITIONS, PAIN IS THEIR
BIGGEST CONCERN AND COMPLAINT.
AS WE ALL EITHER KNOW OR ARE
LEARNING, PAIN INCREASES IN
PREVALENCE WITH AGE.
SO I'M GOING TO PICK UP WHERE
MICHAEL LEFT OFF AT THE BOTTOM
OF THIS.
THE CHEVRON IS SLIGHTLY
DIFFERENT COLORS ON THIS SLIDE,
BUT IT'S BASICALLY GIVING YOU AN
OUTLINE OF THE HEAL PROGRAMS IN
THE SAME WAY.
SO THE CLINICAL TEAM PICKS UP
WITH THE BIOMARKER PROGRAMS AND
MOVES FROM THERE ON THE CHEVRON
TO THE BIG LARGE BACKPACK OR THE
BACK PAIN CONSORTIUM, AND THEN
WE HAVE THREE CLINICAL NETWORKS,
ONE IS EPPIC NET, ONE IS THE
PRAGMATIC TRIALS, EFFECTIVENESS
AND IMPLEMENTATION.
I SKIPPED OVER THE HOPE KIDNEY
DISEASE TRIAL.
THAT IS ONE LARGE NATIONWIDE
CLINICAL TRIAL THAT IS LOOKING
AT ALTERNATIVES TO OPIOIDS FOR
PEOPLE WHO ARE EXPERIENCING
SEVERE PAIN DURING KIDNEY
DIALYSIS, WHICH THEY NEED END
STAGE RENAL DISEASE.
AND THEN THERE ARE TWO NEW
PROGRAMS AT THE BOTTOM RIGHT
THAT JUST STARTED UP THIS YEAR.
THE HEALTH EQUITIES PROGRAM, AND
THE PAIN -- AND A PROGRAM TO
INTEGRATE CARE FOR THOSE WITH
PAIN AND OPIOID USE DISORDER OR
ARE AT RISK FOR OPIOID USE
DISORDER.
SO I'LL TELL YOU A LITTLE BIT OF
THE HIGHLIGHT AND SOME OF THE
PROGRAMS WE HAVE LISTED HERE,
AND THEN A LITTLE BIT MORE
DETAIL ON THE NEW PROGRAMS
BECAUSE THE ADVISORY COMM COMMIE
HASN'T HEARD ABOUT THEM YET.
HEAL IS ALSO DOING SOME REALLY
IMPORTANT CROSS-CUTTING WORK
THAT INCLUDES EFFORTS THAT WILL
HELP ALL OF THESE PROGRAMS.
ONE IS COMMUNITY ENGAGEMENT.
WE'LL TALK ABOUT THAT A LITTLE
BIT MORE.
WORKFORCE ENHANCEMENT ON THE
CLINICAL PAIN RESEARCH SIDE ALSO
HAS SOME NEW PROGRAMS IN
ADDITION TO THOSE THAT MICHAEL
MENTIONED.
WE'RE WORKING TOWARD ENHANCING
THE DIVERSITY OF THE CLINICAL
COHORT AS WELL AS THE WORKFORCE.
DATA HARMONIZATION HAS BEEN A
BIG HUGE EFFORT ACROSS ALL THESE
HEAL CLINICAL TRIALS IN BOTH
PAIN AND OUD TO MAKE SURE THAT
THE DATA ARE HARMONIZED AND
TRANSLATABLE ONCE THE STUDIES
HAVE BEEN FINISHED.
SO IN THE NEXT SLIDE, I WANT TO
START A LITTLE BIT OF THE
EVOLUTION OF THE BIOMARKER
PROGRAM.
THE EARLY PROGRAM WAS LED BY
MARYANN PELAMONTER.
WE FUNDED EIGHT AWARDS, LOOKING
AT PROGR PROGNOSTIC, SEVEN PROGC
AND ONE DIAGNOSTIC BIOMARKER
THAT WOULD BE IN THE DEVELOPMENT
AND VALIDATION STAGES TO HELP
MONITOR DISEASE PROGRESSION OR
DIAGNOSIS OF A PARTICULAR PAIN
CONDITION.
SO THESE COVER EIGHT DIFFERENT
PAIN CONDITIONS, AND THEY WILL
BE MOVING INTO THEIR CLINICAL
VALIDATION STAGE --
>>WE JUST LOST YOUR SOUND.
>>UH-OH.
>>WE DIDN'T LOSE HER SOUND.
WALTER, WE DIDN'T LOSE HER
SOUND.
>>WE CAN HEAR HER VERY WELL.
>>IT'S A PROBLEM IN YOUR
HEADPHONES.
>>CAN MOST OF YOU HEAR ME?
>>YES.
KEEP GOING.
>>ALL RIGHT.
SO I WILL KEEP GOING.
AND HOPEFULLY WALTER WILL GET
SOUND BACK.
THE PROGRAM WAS REALIGNED BASED
ON THE RECOGNITION THAT WE
REALLY ALSO NEEDED BIOMARKERS
THAT COULD BE MORE CLOSELY
ALIGNED WITH EARLY -- ESPECIALLY
EARLY CLINICAL TRIALS, EARLY
PHASED, BUT BE USED IN ALL
CLINICAL TRIALS TO FACILITATE
AND IMPROVE THE OUTCOMES OF THE
TRIAL.
SO THE NEW PROGRAM WAS DEVELOPED
AND IS LED BY RAM ARUDSHONDRUM
AND IT IS CALLING FOR
APPLICATIONS THAT WOULD LOOK FOR
DEVELOPMENT AND VALIDATION OF
BIOMARKERS THAT WOULD BE
PREDICTIVE AND ABLE TO MON OR
TREATMENT RESPONSES.
SO WE COULD THEN STRATIFY
PATIENTS BETTER INTO CLINICAL
TRIALS FOR A MORE LIKELY
FEASIBLE OUT COME FROM THOSE
TRIALS.
AND A NEW PROGRAM JUST LAUNCHED
THIS YEAR IS TO LOOK AT
BIOMARKERS IN MYOFASCIAL
TISSUES.
MYOFASCIAL TISSUES WOULD INCLUDE
MUSCLE FASCIA AND OTHER
ASSOCIATED TISSUES FOR WHICH
MEANL PAIN CONDITIONS ARE LIKELY
TO HAVE A CONTRIBUTION FROM
THESE PARTICULAR TISSUES,
ESPECIALLY THOSE WITH HIGH
PREVALENCE, LIKE LOW BACK PAIN,
NECK PAIN, ET CETERA.
SO THIS PROGRAM IS LOOKING FOR
TOOLS, NEW TECHNOLOGIES THAT
COULD LOOK AT THE MECHANICAL
PROPERTIES, THE STRUCTURE AND
THE PHYSIOLOGICAL PROPERTIES OF
LATENT OR HEALTHY, IF YOU WILL,
MYOFASCIAL TISSUE AND ALSO BE
ABLE TO ASSESS CHANGES IN THOSE
TISSUES ONCE PAIN HAS SET IN.
SO SEVEN PROJECTS HAVE JUST
LAUNCHED HERE, AND WE HOPE THAT
THEY WILL ALL TRANSITION TO BE
SUCCESSFUL IN PHASE TWO.
THE NEXT SLIDE IS A LITTLE BIT
OF AN UPDATE ON THE BACKPACK
CONSORTIUM.
THE BAC PAC CONSORTIUM IS A
LARGE COMPREHENSIVE AND
COLLABORATIVE SET OF DIFFERENT
TYPES OF PROGRAMS THAT WOULD
FEED INTO DEVELOPMENT OF
DIAGNOSTIC TOOLS AND ALSO THE
BEST APPROACHES TO DEEP
PHENOTYPING PATIENTS WITH
DIFFERENT TYPES OF BACK PAIN IN
ORDER TO DEVELOP ALGORITHMS OF
HOW THOSE TOOLS CAN BE USED TO
PREDICT OPTIMAL TREATMENT FOR
PEOPLE WITH DIFFERENT KINDS OF
BACK PAIN, AND FOR THEIR OWN
INDIVIDUAL CHARACTERISTICS.
SO THE EXCITING FUSE HERE IS
THAT ALL THESE PIECES ARE
BEGINNING TO COME TOGETHER TO
THE BAC PAC IS LAUNCHING A TRIAL
CALLED THE BES TRIAL, BIOMARKERS
FOR EVALUATING SPINE TREATMENTS.
IT'S A SMART DESIGN TRIAL WHERE
400 PATIENTS ARE BEGINNING TO BE
ENROLLED IN ORDER TO TAKE THEIR
PARTICULAR PHENOTYPES, USE THE
TOOLS THAT WE'VE GOT FOR
ASSESSMENT, AND DIAGNOSTICS, AND
THEN RUN THEM THROUGH DIFFERENT
TREATMENTS THAT THEY COULD SORT
OF SEQUENTIALLY GO THROUGH
DIFFERENT TREATMENTS TO SEE WHAT
WORKS BEST FOR WHOM, AND
OTHERWISE REALLY DEVELOPING SOME
TAILORED PERSONALIZED TREATMENTS
FOR LOW BACK PAIN, WHICH HAS
BEEN A VERY CHALLENGING AREA OF
PAIN MANAGEMENT.
ON THE NEXT SLIDE, WE'LL GO TO
ONE OF OUR CLINICAL TRIAL
NETWORKS WHICH IS HOUSED AT
NINDS UNDER THE LEADERSHIP OF
BARBARA KARP AND HER TEAM, AND
THIS IS THE EPPIC NET OR THE
EARLY PHASE INVESTIGATIONAL
TRIAL NETWORK.
THIS IS FOR PHASE TWO TRIALS
WITH ASSETS THAT HAVE COME
THROUGH THEIR FOUR STAGES OF
REVIEW, AND ACTUALLY GOTTEN TO
THE POINT WHERE NIH HAS INVESTED
IN A TRIAL USING AN ASSET
SUBMITTED BY THE OUTSIDE
COMMUNITY AND WE ACTUALLY
DEVELOPED A PROTOCOL FOR THEM
WITH OUR INVESTIGATIVE TEAM, AND
RUN THAT TRIAL THROUGH THE
EPPICNET NETWORK, WHICH IS
NATIONWIDE.
SO ONE TRIAL, KNEE
OSTEOARTHRITIS, IS RECRUITING.
THIS IS USING A SELECTIVE
ANTAGONIST SO THE CHEMOKINE
PATHWAYS FOR NEOOSTEOARTHRITIS.
THE OTHER TRIAL FOR PAINFUL
DIABETIC NEUROPATHY, WE EXPECT
WE'LL HAVE MORE OF THESE TRIALS
IN THE LIFETIME OF EPIC NET SO
WE'RE USING A PLATFORM PROTOCOL
DEVELOPED BY OUR INVESTIGATORS
FOR THIS TYPE OF PAIN, AND THIS
ONE HAS JUST LAUNCHED AND IT
WILL BE LOOKING FOR A NATURAL
SMALL MOLECULE FROM A PLANT
BACTERIUM, AND WHICH THE THIS
MECHANISM IS NOT COMPLETELY
KNOWN BUT THAT HAS JUST
LAUNCHED.
SO EXCITING, THE NETWORK IS UP
AND RUNNING AND THAT THESE TWO
TRIALS ARE ACTUALLY IN
RECRUITMENT PHASES.
AND MY NEXT UPDATE IN THE NEXT
SLIDE IS ON THE COMPARATIVE
EFFECTIVENESS RESEARCH NETWORK,
WHICH WAS ESTABLISHED BY HEAL TO
RUN LARGE COMPARATIVE
EFFECTIVENESS RESEARCH TRIALS,
AND WE CURRENTLY HAVE FUNDED
EIGHT TRIALS, AND YOU CAN SEE
THAT THESE TRIALS HAPPEN IN
DIFFERENT SETTINGS, THEY ARE
OCCURRING IN POPULATIONS ACROSS
THE LIFESPAN, AND THEY ARE A
NUMBER OF TRIALS THAT LOOK AT
DIFFERENT PAIN CONDITIONS, AND
ARE USING EITHER OR BOTH OF
PHARMACOLOGICAL AND
NON-PHARMACOLOGICAL
INTERVENTIONS THAT ARE BEING
TESTED.
THE EXCITING NEWS ON THIS ONE IS
THAT DESPITE COVID AND ALL THE
SLOW-DOWNS, WE HAVE A TRIAL
LOOKING AT POSTOPERATIVE PAIN IN
C-SECTIONS, RECOGNIZING THE FACT
THAT 1 IN 300 C-SECTION MOMS GO
ON TO DEVELOP OPIOID USE
DISORDER BECAUSE OPIOID ARE VERY
TYPICALLY PRESCRIBED TO TAKE
HOME AFTER A C-SECTION.
SO THEY COMPLETED ENROLLMENT IN
TWO YEARS, AND WE'LL BE ACTUALLY
PRESENTING SOME DATA NEXT
WEEKEND -- NEXT WEEK, RATHER, IN
AN OPEN FORUM.
SO THE TRIAL REALLY MOVED ALONG
QUICKLY.
THE HOPE IS THAT GIVING THESE
MOTHERS ALTERNATIVES TO OPIOIDS
AFTER THE CESAREAN SECTION WILL
REDUCE THE NUMBER WHO GO ON TO
DEVELOP OPIOID USE DISORDER.
NEXT SLIDE WILL TELL YOU THE
OVERVAWOVERVIEW OF THE STUDIES E
INCLUDED IN OUR PRAGMATIC
IMPLEMENTATION TRIALS, AND THESE
ARE STILL ONGOING, STILL
ENROLLING, SO WE DON'T HAVE ANY
OUTCOMES REPORTS FOR YOU AT THIS
POINT, BUT THE IDEA HERE IS THAT
YOU CAN SEE WHAT PAIN CONDITIONS
ARE BEING STUDIED, AND WHAT
INTERVENTIONS ALL BEING
NON-PHARMACOLOGICAL, WHICH WAS
THE INTENT OF THE PROGRAM, IN
LARGE HEALTHCARE SYSTEMS IN A
REAL WORLD SETTING.
THE NEXT SLIDE, I'M GOING TO
SHIFT GEARS HERE A LITTLE BIT
AND THEN COME BACK BRIEFLY TO
THE NEW PROGRAMS THAT I
MENTIONED.
AND WHAT WE THOUGHT WAS
IMPORTANT WAS TO TAKE A CAREFUL
LOOK AT THE PORTFOLIO OF GRANTS
THAT WE ALREADY HAVE IN THE
CLINICAL PAIN MANAGEMENT REALM,
CLINICAL RESEARCH REALM HERE,
AND SO ANDERSONS AND ANTHONY
DOMINICALE FROM OUR OFFICE HERE
HAVE PULLED DATA FROM ALL OF THE
CLINICAL TRIALS AND LONGITUDAL
STUDIES ACROSS HEAL FOR PAIN,
AND LOOKED AT WHAT PAIN
CONDITIONS ARE BEING STUDIED AND
AT WHAT FREQUENCY AS MEASURED
PER NUMBER OF GRANT.
AND SOME GRANTS WILL HAVE MORE
THAN ONE TRIAL EMBEDDED IN THEM,
BUT EACH TIME A PAIN CONDITION
IS MARKED HERE, IT'S ONLY MARKED
ONCE.
SO FOR EXAMPLE, BACK PAIN IS NOT
INCLUDED IN CHRONIC PAIN, THESE
ARE SEPARATE APPLICATION.
YOU CAN SEE HERE THAT BACK PAIN
IS VERY WELL REPRESENTED ACROSS
THE HEAL PORTFOLIO, BUT THAT
MANY OTHER PAIN CONDITIONS ARE
BEING CONSIDERED EITHER FOR
BIOMARKER DEVELOPMENT OR FOR THE
MOST PART THESE ARE ALL CLINICAL
TRIALS, A FEW LONGITUDINAL
OBSERVATIONAL TRIALS ARE
INCLUDED IN THIS DATA.
THE NEXT SLIDE IS A LITTLE BIT
MORE INFORMATION ON THE ANALYSIS
THAT THEY DID, LOOKING AT THE
KINDS OF INTERVENTIONS THAT ARE
BEING TESTED IN THESE CLINICAL
TRIALS.
AND THESE ARE SUBCATEGORIES THAT
WERE COMBINED.
SO FOR EXAMPLE, THE BEHAVIORAL
HEALTH LINE WHICH IS HIGHLY
PREDOMINANT ACROSS THE NUMBER OF
TRIALS AS BEING INCLUDED AS AN
INTERVENTION WOULD INCLUDE
THINGS LIKE COGNITIVE BEHAVIORAL
THERAPY, MINDFULNESS, EDUCATION
AND SO ON.
NON-INTERVENTION IS
REPRESENTATIVE MOSTLY OF THE
PHENOTYPIC FUNCTIONS IN THE
OBSERVATIONAL TRIALS.
AND THE LAST GRANT ON GRAPH ON T
HERE, WE KNOW PAIN CARE IS
DELIVERED IN DIFFERENT SETTINGS,
SO IT'S IMPORTANT TO KNOW THAT
OUR RESEARCH SETTINGS ARE
REPRESENTED -- REPRESENTATIVE OF
WHERE CARE IS BEING PROVIDED.
SO THIS GIVES US AND GIVES YOU A
SENSE OF WHERE THESE STUDIES ARE
HAPPENING IN DIFFERENT TYPES OF
SETTINGS AND WITHIN DIFFERENT
TYPES OF CLINICS, FOR EXAMPLE.
,
AS APPROPRIATE TO THE STUDY.
WE DUG A LITTLE BIT DEEPER INTO
BACK PAIN BECAUSE THAT'S ONE OF
THE MOST PREVALENT CONDITIONS IN
THE WORLD, 39% OF ADULT HAD BACK
PAIN IN THE LAST THREE MONTHS,
ACCORDING TO A U.S. SURVEY OF
ADULTS.
AND SO WA WE'VE OPENED UP THE
SUBCATEGORIES HERE AND LOOKED AT
WHERE BACK PAIN STUDIES ARE
HAPPENING, SO THAT'S OUR STUDY
SETTING AND IT INCLUDES A NUMBER
OF DIFFERENT AND APPROPRIATE
HEALTHCARE SETTINGS THAT RUN
FROM TELEHEALTH ALL THE WAY TO
HEALTH SYSTEMS, COULD BE
INPATIENT CARE, INCLUDING
FEDERALLY QUALIFIED HEALTHCARE
SETTINGS WHICH TYPICALLY SERVICE
PEOPLE WHO DEAL WITH HEALTH
DISPARITY.
INTERVENTIONS AS YOU CAN SEE ON
THE RIGHT-HAND SIDE ARE BROAD,
AND SO A LOT OF DIFFERENT THINGS
ARE BEING LOOKED AT, IN PART
THROUGH THE BAC PAC PROGRAM BUT
ALSO THROUGH OTHER PARTS OF THE
HEAL CLINICAL PAIN MANAGEMENT
PROGRAM.
ON THE NEXT SLIDE ASKS THE
QUESTION, AND THIS IS WHY I HELD
OFF ON TELLING YOU WHAT THE NEW
PROGRAMS ARE ABOUT, THEY HELP US
TO ANSWER SOME OF THESE
QUESTIONS.
SO WHY IS PAIN MANAGEMENT SO
CHALLENGING?
FIRST OFF, REIMBURSEMENT IS
OFTEN DIFFICULT TO GET,
ESPECIALLY FOR CHRONIC PAIN OVER
TIME.
CMS IS MAKING MAJOR CHANGES IN
THEIR MEDICARE REIMBURSEMENT
STRATEGIES IN GIVING CHRONIC
PAIN SORT OF A LONGER PERIOD OF
REIMBURSEMENT AND A BROADER
ACCESS TO DIFFERENT TYPES OF
CARE THAT WOULD FALL UNDER NEEDS
FOR PEOPLE WITH CHRONIC PAIN.
SO THAT'S PRETTY EXCITING.
ACCESS TO CARE, WE ARE LOOKING
AT THE DEMOGRAPHICS OF WHERE
THESE STUDIES HAPPEN.
SOME OF THE ONES THAT ARE
ONGOING ARE TARGETED TO RURAL
AREAS.
THERE'S A NEW PROGRAM THAT WILL
BE FUNDED IN 2023 BUT IT HAS NOT
YET, LOOKING AT GRANTS
SPECIFICALLY TRYING TO IMPROVE
PAIN MANAGEMENT IN RURAL
POPULATIONS.
DISPARITIES, WE'LL COME TO THE
HEALTH EQUITIES PROGRAM IN A FEW
MINUTES, BUT BIAS AND A STIGMA
ARE OFTEN PART OF THE REASON
THAT PEOPLE WHO EXPERIENCE
HEALTH DISPARITIES DON'T GET
GOOD PAIN CARE.
TRAINING AND MENTORING, I THINK
MICHAEL GAVE YOU A REALLY CLEAR
SENSE OF -- AND WALTER'S
PRESENTATION OF WHY WE NEED
BETTER TRAINING, BETTER
MENTORING EXPERIENCE TO GET
PEOPLE AT EARLIER CAREER STAGES
TO COME IN AND STAY IN PAIN
RESEARCH.
COMPLEXITY OF PAIN IS CLEAR.
WE HAVE OTHER CHRONIC
CONDITIONS, SOMETIMES MULTIPLE
PAIN CONDITIONS, DIFFERENT
PATHWAYS, MECHANISMS FOR PAIN
MAKES RESEARCH AND CLINICAL
APPROACHES EVEN MORE DIFFICULT,
I THINK TO SOME EXTENT THE BACK
PAIN TRIALS ARE GOING TO HELP
WITH THIS A BIT AS FAR AS
DEVELOPING PERSONALIZED MEDICINE
TECHNIQUES.
LIFESPAN, AGE CHANGES, HOW WE
EXPERIENCE PAIN AND HOW PAIN IS
ACTUALLY MANIFESTED OVER TIME.
AND THE NEXT SLIDE, WE HAVE SORT
OF THE SAME PROBLEM AREAS
LISTED.
THE COMPLEXITY OF PAIN IS --
CO-OCCURRING CONDITIONS.
HEALTH DISPARITIES, OUR HEALTH
EQUITIES PROGRAM WILL BEGIN TO
ADDRESS THAT.
TRAINING AND MENTORING,
WORKFORCE, I'LL TALK ABOUT IN A
FEW MINUTES.
AND I'VE ALREADY MENTIONED
ACCESS TO CARE WITH THE RURAL
HEALTH PROGRAM COMING UP, AND
REIMBURSEMENT WITH CMS BEING A
HUGE CONTRIBUTOR HE HERE TO
IMPROVING THE SITUATION.
SO THE NEXT SLIDE TELLS YOU A
LITTLE BIT, TINY BIT, ABOUT THE
PROGRAM LOOKING AT INTEGRATIVE
CARE FOR PEOPLE WHO HAVE CHRONIC
PAIN AND OUD OR ARE AT RISK FOR
OUD BECAUSE OF THE OPIOIDS THAT
THEY'VE BEEN USING OR THE TIME
FRAME THEY'VE BEEN USING THEM,
ET CETERA, ET CETERA.
AND SO WE HAVE FUNDED FIVE
AWARDS IN THIS REALM, THERE'S A
SECOND ROUND COMING OUT, WE HOPE
THIS SUMMER, THAT'S UNDER REVIEW
THAT WILL ADD TO THIS PART OF
THE PORTFOLIO.
AND ON THE RIGHT IS A LITTLE BIT
OF DETAIL FROM OUR PORTFOLIO
ANALYSIS THAT SHOWS YOU THE
POPULATIONS THAT WOULD BE
INCLUDED IN THESE STUDIES.
SO THERE ARE MORE POPULATIONS
HERE THAN THERE ARE TRIALS.
BECAUSE EACH OF THESE AWARDS HAS
MULTIPLE TRIALS EMBEDDED IN THE
AWARD.
SO FOR SOME OF THESE, PATIENTS
WHO COME IN TO THE STUDY HAVE TO
BE ON MEDICATION-ASSISTIVE
TREATMENT FOR OUD OR BE AT RISK
FOR OUD, SO THEY MAY BE ON
OPIOID, FOR EXAMPLE, AND SO THE
INTERVENTIONS HERE ARE, I THINK,
BOTH PHARMACOLOGICAL AND
NON-PHARMACOLOGICAL, AND I THINK
APPROPRIATELY TARGETED TO THE
DIFFERENT POPULATIONS THAT ARE
COMING IN THROUGH THIS SET OF
STUDIES.
A LOT OF THESE ARE BEHAVIORAL,
AND ARE BEING -- PARTLY BECAUSE
THEY CAN BE ADMINISTERED IN THE
SETTINGS WHERE THE PATIENT
COHORT IS FOUND, FOR EXAMPLE,
THROUGH OUD CLINICS OR PRIMARY
CARE, THE V.A. IS SUPPORTING ONE
OF THESE TRIALS.
SO THIS IS AN AREA WHERE PAIN
COMPLEXITY MAKES MANAGEMENT
VERY, VERY DIFFICULT, AND WE
HOPE TO GET SOME GOOD ANSWERS
FROM THIS ONE.
ON THE NEXT SLIDE IS A LAST ONE
ON THE NEW PROGRAMS, NEW
RESEARCH PROGRAMS, THAT IS
DEALING WITH HEALTH DISPARITIES
IN PEOPLE WHO HAVE PAIN OR PAIN
WITH CO-OCCURRING HEALTH
CONDITIONS C.
FOR MOST OF THESE, THE TARGET IS
MENTAL HEALTH, ANXIETY,
DEPRESSION, ET CETERA, AND MOST
OF THESE STUDIES THAT ARE
DEALING WITH PAIN AND
CO-OCCURRING HEALTH CONDITIONS.
SO BIAS AND STIGMA CAN HAPPEN AT
A NUMBER OF DIFFERENT LEVELS,
AND WHAT WE WANTED TO DO THROUGH
THIS PROGRAM, WHICH IS WRUN BY
SHARISE SRINKAR AT NINDS, IS
INCLUDE DIFFERENT LEVELS OF
RESEARCH IN THESE AWARDS THAT
CAN HELP TO REDUCE STIGMA AND
BIAS, EITHER AT THE
PROVIDER-PATIENT LEVEL OR ALL
THE WAY UP TO THE SYSTEMS LEVEL
WHERE THERE MAY BE BIAS AT THE
SYSTEMS LEVEL THAT IS KEEPING
FOLKS FROM GETTING APPROPRIATE
PAIN CARE.
SO THE POPULATION ON THE FAR
LEFT COLUMN, I THINK GIVES YOU A
GOOD IDEA THAT THERE'S A BROAD
RANGE OF POPULATIONS THAT
EXPERIENCE HEALTH DISPARITIES
INCLUDED IN THIS PART OF THE
PORTFOLIO, AND PT PAIN
CONDITIONS ARE LISTED IN THE
ORANGORANGEY ONE, THE ORANGEY C,
AND AGAIN, A LARGE RANGE OF
DIFFERENT TYPES OF PAIN WILL BE
STUDIED.
AND THE INTERVENTIONS HERE
INCLUDE A LOT OF SELF MANAGEMENT
AT THE PATIENT PROVIDER LEVEL,
SOME IS COACHED, AND ALL ARE
CULTURALLY ADAPTED FOR THE
POPULATION OF STUDY, AND SOME OF
THESE ARE ALSO ADOPTIVE AT THE
SYSTEMS HEALTHCARE LEVEL, WHICH
IS THE LAST COLUMN ON HERE.
AGAIN, THE SETTINGS ARE
APPROPRIATE TO THE STUDY AND TO
THE POPULATION BEING STUDIED.
AND THEN THE NEXT SLIDE GIVES
YOU A REALLY QUICK OVERVIEW OF
WHAT THE APPROACH IS TO
ENHANCING THE CLINICAL PAIN
RESEARCH WORKFORCE, AND SO I
THINK THERE'S A PARTICULAR NEED
HERE.
A LOT OF FOLKS IN THIS AREA ARE
RETIRING OR AREN'T REALLY HAVING
THE TIME TO MENTOR ANYMORE, SO A
LOT OF DIFFERENT PROGRAMS TO
BEEF UP THE WORKFORCE, SOME I
THINK ARE REALLY NOVEL AND
INNOVATIVE.
WE'VE GOT SUPPLEMENTS TO SUPPORT
EARLY CAREER TRAINEES AND WE'VE
GOT AWARDS TO PROTECT MENTORS'
TIME TO DEVOTE TO MENTORING,
THAT'S THE K-24, AND TWO LINKED
NEW PROGRAMS, WHICH WE'RE REALLY
EXCITED ABOUT, A K12 WHICH IS A
TRAINING PROGRAM FOR EARLY
CAREER STAGE INVESTIGATORS
HOUSED AT UNIVERSITY OF MRI, BUT
ITMICHIGAN, BUT IT WILL TAKE IN
TRAINEES ACROSS THE NATION.
SO IT WILL BE NOT ONLY MICHIGAN
FOLKS BUT WILL BE NATION-WIDE,
AND THEY ARE LINKED AND WORKING
CLOSELY WITH THE R-24 AWARDEES,
WHICH IS A COORDINATING CENTER
FOR -- ACTUALLY THIS ONE IS
REALLY EXCITING.
IT A COORDINATING CENTER THAT
WILL SET UP A PLATFORM OR HAS
SET UP A PLATFORM FOR NIH,
BEYOND HEAL, NIH AWARDEES AT THE
EARLY CAREER STAGE OF
DEVELOPMENT TO GATHER AT AN
ANNUAL MEETING -- SORRY, THE
PLATFORM WILL BE FOR THEM TO
NETWORK, TO MEET PEOPLE, TO
COLLABORATE, TO LEARN, TRAINING
RESOURCES, ET CETERA, ET CETERA,
DEVELOPED TOGETHER WITH THE
UNIVERSITY OF MICHIGAN.
AND THESE TWO PROGRAMS ARE RUN
BY -- THE ADD IN MY OPINION
SUPPLEMENTS ARE RUN BY REBECCA
HUMMER.
AND WE'RE HOPING TO BEGIN TO
TARGET EARLIER STAGES OF
TRAINEES WITH SOME OF OUR
PROGRAMS AT THE POSTDOC LEVEL.
AND THEN THE LAST SLIDE IS --
IT'S A SMALL SLIDE ABOUT A
REALLY, REALLY BIG EFFORT ON THE
PART OF HEAL TO ENGAGE THE
COMMUNITY AND HOW WE MAKE -- WE
CAN MAKE OUR CLINICAL STUDIES
MORE MEANINGFUL TO THE PEOPLE
WHO THOSE STUDIES ARE BEING DONE
TO SUPPORT.
AND SO WE HAVE A HEAL COMMUNITY
PARTNER COMMITTEE THAT IS --
THAT INCLUDES PEOPLE WITH LIVED
EXPERIENCE OF OPIOID USE
DISORDER, SUBSTANCE USE
DISORDER, CHRONIC PAIN AND/OR
BOTH, AND FAMILY MEMBERS AND
COMMUNITY ADVOCATES IN THAT
PARTICULAR AREA.
AND THEIR JOB IS REALLY TO HELP
US SET UP TRIALS THAT INCLUDE
THE VOICES OF THESE PEOPLE AND
MAKE THEM MORE MEANINGFUL, SO
THE PICTURE ON THE RIGHT IS
MEANT TO REPRESENT THE NEED OF
THE INVESTIGATORS WHO ARE
RUNNING THESE TRIALS TO HAVE
INPUT FROM THE OUTSIDE
COMMUNITY, IF YOU WILL, OUR
COMMUNITY, INTEGRATE THEM INTO
THEIR VOICES, AND THEIR IDEAS
AND THEIR THOUGHTS INTO HOW WE
RUN THE TRIALS.
SO IT BEEN A REALLY, REALLY
WONDERFUL EXPERIENCE TO TALK
WITH THEM AND HEAR THEIR VOICES,
AND TO HELP IN WAYS SUCH AS HOW
WE WOULD WANT TO SUPPORT -- GIVE
MORE RESOURCES TO SUPPORT THE
HEAL AWARDEES IN ORDER TO MAKE
SURE THEY CAN DO A REALLY GOOD
JOB WITH COMMUNITY ENGAGEMENT
AND DIVERSITY OF THE POPULATION
AS THEY GO FORWARD WITH THE
TRIALS, AND PART OF THAT, WHAT
WE'VE DONE IN THE BACKGROUND, IS
AT A SUGGESTION OF THE HEAL
COMMUNITY PARTNER COMMITTEE, IS
TO DO A BROAD RESEARCH STRATEGY
ACROSS ALL THE HEAL
INVESTIGATORS TO UNDERSTAND WHAT
THEY ARE ALREADY DOING FOR
COMMUNITY ENGAGEMENT AND HOW WE
CAN USE THE INFORMATION RECEIVED
FROM THE SURVEY TO -- AS LESSONS
LEARNED FOR FOLKS WHO MAYBE
AREN'T DOING AS MUCH AS THEY
COULD, OR DON'T KNOW HOW TO DO
IT OR DON'T HAVE THE RESOURCES
TO DO IT.
SO I'M GOING TO SAY THANKS VERY
MUCH FOR THE TIME.
MICHAEL AND I, I THINK HAVE A
LITTLE BIT OF TIME FOR QUESTIONS
AND ANSWERS SO I'LL STOP THERE.
>>WE'RE QUITE BEHIND IN TIME,
WHICH ISN'T YOUR FAULT OR
MICHAEL'S.
SO MAYBE WE'LL TAKE ONE OR TWO
URGENT QUESTIONS AND THEN PEOPLE
CAN ASK QUESTIONS IN THE CHAT
PERHAPS.
ARE THERE ANY QUESTIONS?
BOB?
>>THANK YOU.
GREAT PRESENTATIONS, VERY
IMPORTANT PROGRAM.
BUT I WONDER IF YOU COULD TELL
US SORT OF AT A HIGH ALTITUDE
WHERE THE PROGRAM STANDS IN
TERMS OF DEVELOPING NEW TYPES,
NEW CATEGORIES OF PHARMACOLOGIC
INTERVENTIONS WHICH ARE NOT
OPIOID, BUT WHICH NONETHELESS
ARE AS EFFECTIVE AS OPIATES, AND
HOPEFULLY WHICH ARE NOT
ADDICTIVE, OR IS THAT EVEN
POSSIBLE?
BECAUSE IT SEEMS TO ME WHAT
YOU'VE EMPLOYED HERE IS A
WONDERFUL COMBINATION OF
EXISTING THERAPIES BUT THE
QUESTION IS, ARE THERE NEW
THERAPIES AROUND THE CORNER?
THANK YOU.
>>GREAT QUESTION.
THANKS, LINDA.
SO AS I MENTIONED DURING THE
TALK, WE HAVE 56 TARGETS THAT
ARE BEING IDENTIFIED, NONE OF
THEM ARE OPIOID TARGETS.
ACTUALLY AGONISTS OF THE MU
OPIOID RECEPTOR ARE EXCLUDED
FROM ALMOST ALL OF THE HEAL
PROGRAMS.
SO THOSE 13 DEVELOPMENTAL
PROJECTS THAT YOU SEE IN THE
PTDP, ALL OF THE HUNDREDS OF
COMPANIES THAT HAVE APPLIED TO
THE SBISR01s, THEY'RE ALL
NON-OPIOID OR NON-ADDICTING.
AND AS A PART OF THE MANY OF
THOSE INITIATIVES, YOU HAVE TO
INCLUDE TESTING OF THE ABUSE
LIABILITY IN THEM.
SO ON THAT SIDE, THE WHOLE
PROGRAM IS NON-ADDICTING.
BUT ON THE PART OF YOUR QUESTION
IS HOW CLOSE ARE WE, HOW CLOSE
ARE WE, THAT'S A REALLY
DIFFICULT QUESTION.
SO I THINK THAT IN MY OPINION,
THE CLOSEST PROJECTS ARE THE
SBIR ONES.
IT'S BECAUSE THEY'RE JUST BUILT
FOR COMMERCIALIZATION.
AND I THINK THE SOONEST ONES
THAT COULD MAKE TO THE MARKET
ARE SOME OF THE DEVICE ONES.
BUT WITH THAT SAID, THERE ARE
SOME VERY INTERESTING PROGRAMS
THAT I DIDN'T SUMMARIZE NOW THAT
WE HAD FUNDED -- THEY ORIGINALLY
STARTED WITH THE BPN PROGRAM
THAT WE'D FUNDED WITH HEAL WITH
A SUPPLEMENT PROGRAM, YOU KNOW,
THAT ARE IN CLINICAL TESTING AT
THIS POINT.
AND YOU KNOW, THOSE TARGETS
REALLY SHOW NO ABUSE LIABILITY.
DO YOU THINK IT'S POSSIBILITY?
YES.
POSSIBLE
>>YES.
>>ON THAT AFFIRMATIVE NOTE, ONE
LAST QUESTION FROM TIM, THEN PUT
OTHER QUESTIONS IN THE CHAT.
>>FOR MICHAEL, SOME
CLARIFICATION IN THE CALL FOR
NEW NON-RODENT ANIMAL MODELS
FOR, I GUESS, PRE-CLINICAL PAIN
STUDIES.
DO YOU HAVE AN ALGORITHM OR
CRITERIA, BECAUSE OBVIOUSLY THE
MORE ADVANCED IT GOES TO SAY A
NON-HUMAN PRIMATE, THERE'S MORE
RESTRICTIONS, THERE'S EXPENSE,
ET CETERA, SORT OF MANIPULATIONS
YOU CAN'T DO COMPARED TO A
RODENT.
SO IS THERE SOME KIND OF WHEEL
OF FORTUNE WHERE YOU SORT OF
DECIDED WHAT YOUR NEEDS ARE,
WHAT YOU'RE WILLING TO ACCEPT IN
TERMS OF EITHER THE PRICE OR THE
LIMITATIONS OF THAT MODEL?
>>SO THE FUNDING ANNOUNCEMENT
DOES HAS LIMITS ON THE SIZE OF
THE GRANT ITSELF.
WE'RE HOPING THAT THAT LIMIT IS
WITHIN THE --
>>THIS ISN'T REALLY A FINANCIAL
QUESTION.
IT WAS MORE LITERALLY IN THE
CHOICE OF MODEL, LIKE WHAT THE
CRITERIA WOULD BE TO WHAT WOULD
BE A SUITABLE NON-RODENT MODEL.
AS I SAID, THERE'S GOING TO BE
LIMITS OF WHAT YOU CAN DO --
>>THAT'S THE GOAL OF THE
PROJECT.
BUT THE GOAL OF THE PROJECT IS
TO DEVELOP SOMETHING THAT, FIRST
OF ALL, IN THE FIRST CRITERIA
FOR DETERMINING IF THE MODEL IS
APPROPRIATE IS A SCIENTIFIC ONE.
AND THE SECOND ONE, THEY HAD TO
INCLUDE FEASIBILITY OF
IMPLEMENTING THAT IN A
PRE-CLINICAL SETTING.
SO WE'LL SEE HOW IT SHAKES OUT.
THOSE PROJECTS ARE STILL --
WE'RE WAITING FOR THEM TO COME
IN AND REVIEW THEM, ET CETERA.
>>THANK YOU.
>>OKAY.
I APOLOGIZE FOR RUSHING.
WHY DON'T WE TAKE A 10-MINUTE
BREAK WHICH MEANS COME BACK AT
3:25 SHARP.
THE A.L.S. AGENDA MAY TAKE A
LITTLE LONGER THAN WE THOUGHT SO
I WANT TO STAY NOT TOO FAR
BEHIND SCHEDULE.
I'LL SEAL YOU I SEE YOU IN 10 M.
LET'S GET STARTED.
I WANT TO TRY TO KEEP US NOT TOO
FAR BEHIND.
THE NEXT SESSION, AS YOU CAN
SEE, IS ABOUT STRATEGIC
PRIORITIES FOR A.L.S.
I'M GOING TO TURN IT OVER TO
AMELIE, THE PROGRAM DIRECTOR
FROM THE NEURODEGENERATIVE
CLUSTER, WHO WILL INTRODUCE
THIS.
AMELIE?
>>THANK YOU SO MUCH, AND I HAVE
A SLIDE DECK, SO MAYBE MY SLIDES
CAN BE PULLED UP.
OKAY.
PERFECT.
SO GOOD AFTERNOON, EVERYBODY.
SO IN THE NEXT SESSION, WE ARE
GOING TO DISCUSS STRATEGIC
PRIORITIES FOR RESEARCH
FACILITATED BY NINDS OVER THE
LAST YEAR OR SO.
AND WE'LL ALSO HAVE A
PRESENTATION BY
DR. CORRIGAN-CURAY ABOUT FDA
ACTIVITIES IN RESPONSE TO ACT
FOR A.L.S.
ALSO A PRESENTATION BY WALTER
ABOUT PARTNERSHIP OPPORTUNITIES
IN A.L.S. RESEARCH.
SO THE OVERALL GOAL OF THE
A.L.S. STRATEGIC PLANNING EFFORT
WAS TO IDENTIFY THE HIGHEST
PRIORITIES FOR RESEARCH THAT
WILL LEAD TO THE DISCOVERY OF
BETTER INTERVENTION FOR THE
DIAGNOSIS, TREATMENT,
MANAGEMENT, PREVENTION OR CURE
OF A.L.S.
AND WE STARTED THE PROCESS ABOUT
A YEAR AGO BY RELEASING A
REQUEST FOR INFORMATION TO ASK
FOR INPUT FROM THE PUBLIC ABOUT
THEIR VIEW ABOUT THE TOP
PRIORITIES FOR A.L.S. RESEARCH.
AND IN OUR VIEW, THIS REQUEST
FOR INFORMATION WAS REALLY
SUCCESSFUL.
WE RECEIVED OVER 300 RESPONSES
WITH REALLY VALUABLE FEEDBACK,
AND THESE RESPONSES WERE THEN
SHARED WITH THE STEERING
COMMITTEE THAT WAS LEADING THE
EFFORT AS WELL AS FIVE WORKING
GROUPS.
SO LAST SPRING, THE STEERING
COMMITTEE HELPED US FORM FIVE
WORKING GROUPS THAT WERE TASKED
TO ADDRESS DIFFERENT AREAS OF
A.L.S. RESEARCH, INCLUDING
BASIC, TRANSLATIONAL, CLINICAL
AND QUALITY OF LIFE RESEARCH, AS
WELL AS OPPORTUNITIES FOR
PARTNERSHIPS AND COLLABORATION,
AND DEVELOP AND ARTICULATE
PRIORITY FOR EACH OF THESE AREAS
OF RESEARCH, AND WE ASKED THEM
TO HONE DOWN AND FOCUS ON THREE
PRIORITIES EACH, WHICH WAS
CHALLENGING BECAUSE THEY ALL HAD
A LOT OF GROUND TO COVER.
SO THE WORKING GROUPS THEN MET
VIA TELECONFERENCES MANY TIME
OVEOVER THE SPRING AND THE SUMMR
AND THEY DEVELOPED DRAFT
PRIORITIES WHICH WERE THEN
PRESENTED AND DISCUSSED AT A
PUBLIC WORKSHOP THAT WAS HELD
VIRTUALLY AT THE END OF OCTOBER.
AND DURING THAT WORKSHOP, THERE
WAS A LOT OF OPPORTUNITY FOR
DISCUSSION AND PUBLIC COMMENT.
SO AFTER THE WORKSHOP, THE
REVISED PRIORITIES WERE THEN
POSTED ONLINE FOR ABOUT A MONTH
FOR FORMAL WRITTEN PUBLIC
COMMENT, AND AGAIN, WE GOT
REALLY VALUABLE FEEDBACK AND
THAT FEEDBACK WAS SHARED WITH
THE WORKING GROUP AND THE
STEERING COMMITTEE FOR FURTHER
CONSIDERATION.
AND TODAY, WE ARE SHARING AND
PRESENTING THE FINAL VERSION OF
THESE PRIORITIES TO COUNCIL FOR
CONSIDERATION AND A VOTE.
SO WE REALLY WANT TO THANK ALL
OF THE PARTICIPANTS OF THIS
PLANNING EFFORT.
IT WAS HIGHLY INTERACTIVE.
AS YOU CAN SEE MANY PEOPLE WERE
INVOLVED, SO WE HAD THE STEERING
COMMITTEE, THE WORKING GROUPS
AND IN EACH OF THE WORKING
GROUPS, WE HAD A PERSON LIVING
WITH THE DISEASE OR AFFECTED
WITH THE DISEASE CONTRIBUTE.
WE ALSO HAD STAFF FROM NINDS
INVOLVED.
CHRISTINE WAS OUR OVERALL
COORDINATOR, AS WELL AS
COLLEAGUES FROM OTHER INSTITUTES
AND CENTERS AT NIH, AS WELL AS
THE FDA, THE CDC AND ALSO THE
A.L.S. RESEARCH PROGRAM OF DOD,
AND WE ALSO REALLY WANT TO THANK
THE BROADER A.L.S. COMMUNITY
INCLUDING PEOPLE AFFECTED BY THD
OTHER STA STAKEHOLDERS FOR THEIR
REALLY ACTIVE ENGAGEMENT
THROUGHOUT THE PROCESS.
AND FOR THE DISCUSSION THIS
AFTERNOON, WE HAVE FIVE
PARTICIPANTS, INCLUDING DR. RITA
SATTLER.
SHE IS A PROFESSOR AT THE BARROW
NEUROLOGICAL INSTITUTE, DR. BOB
BROWN, WHO IS THE DIRECTOR OF
THE PROGRAM IN NEUROLOGICAL
THERAPEUTICS AT UMASS CHAN
MEDICAL SCHOOL.
DR. LAYNE OLIFF, WHO IS A PERSON
LIVING WITH A.L.S.
HE IS A RESEARCH AMBASSADOR AND
ALSO AN ADVOCATE FOR THE
INTERNATIONAL ALLIANCE FOR
ALS/MND ASSOCIATIONS.
ELLEN PHILLIPS, CAREGIVER, BOARD
CHAIR EMERITUS OF THE A.L.S.
ASSOCIATION GREATER PHILADELPHIA
CHAPTER, AND ALSO DAN DOCTOROFF,
WHO ALSO LIVES WITH THE DISEASE,
HE'S THE FORMER CHAIRMAN AND CEO
OF SIDEWALK LABS AND THE FOUNDER
AND NOW SERVES ON THE BOARD OF
DIRECTORS OF TARGET A.L.S.
SO NOW I'LL TURN IT OVER TO RITA
AND BOB.
THANK YOU.
>>THANK YOU, AMELIE, AND THANK
YOU TO THE COUNCIL AND THE
MEMBERS OF THE COUNCIL FOR
GIVING US AN OPPORTUNITY TO
SUMMARIZE REALLY THIS INCREDIBLE
EFFORT THAT WAS PUT TOGETHER
OTHER THE LAST YEAR BY, AS YOU
SAW, A REALLY BROAD GROUP OF
PEOPLE AND WE'RE REALLY EXCITED
THAT WE WERE ABLE TO INTEGRATE
THE A.L.S. COMMUNITY
PARTICULARLY IN THIS EFFORT,
INCLUDING PEOPLE LIVING WITH THE
DISEASE, CAREGIVERS OF THE
DISEASE, CARRIERS OF GENETIC
MUTATIONS THAT ARE AT RISK OF
DEVELOPING THIS DISEASE, AND SO
WE'RE REALLY EXCITED TO SHARE
THE RESULTS OF THIS EFFORT WITH
YOU IN THE NEXT FEW MINUTES.
SO IF I CAN HAVE THE FIRST SLIDE
DECK, PLEASE.
THIS IS JUST A SUMMARY OF THE
EFFORTS THAT WE PUT TOGETHER,
AND AS AMELIE NICELY ALREADY
HIGHLIGHTED, WE HAD FIVE WORKING
GROUPS THAT WE PUT TOGETHER FOR
THIS EFFORT.
THE WORKING GROUPS REALLY
FOCUSED ON DIFFERENT ASPECTS
THAT REALLY, IF WE BRING THEM
ALL TOGETHER, SHOULD BE ABLE TO
ADDRESS SOME OF THE CONCERNS AND
THE COMMENTS THAT WE RECEIVED
FROM THE PUBLIC FORUMS TO
ADDRESS QUESTIONS ON WHERE
SHOULD WE REALLY FOCUS ON A.L.S.
RESEARCH IN REGARDS TO THE BASIC
RESEARCH, THE CLINICAL EFFORTS,
AND ALSO THE QUALITY OF LIFE OF
INDIVIDUALS LIVING WITH THE
DISEASE.
SO REALLY JUST TO SUMMARIZE, THE
FIRST WORKING GROUP REALLY
FOCUSED ON ACCELERATING THE
RESEARCH ON THE BIOLOGY BEHIND
A.L.S., AND WE'LL TELL YOU THE
RESULTS OF THE PRIORITIES IN THE
FOLLOWING SLIDES.
WE HAD ONE WORKING GROUP THAT
REALLY SORT OF FOCUSED ON THE
TRANSLATIONAL ASPECTS, HOW DO WE
THEN USE AND UTILIZE WHAT WE
LEARN IN THE RESEARCH AREA AND
HOW DO WE THEN MOVE THIS INTO
THERAPEUTIC DEVELOPMENT.
THE THIRD WORKING GROUP THEN
TOOK ALL OF THAT AND TRIED TO
DEFINE, ONCE WE HAVE A
THERAPEUTIC, HOW DO WE REALLY DO
OUR BEST EFFORT IN RUNNING
CLINICAL TRIALS, HOW DO WE
DESIGN THOSE CLINICAL TRIALS,
WHAT PATIENT POPULATIONS ARE WE
GOING TO INTEGRATE INTO THESE
CLINICAL TRIALS, AND HOW SHOULD
WE GO ABOUT THIS.
AND THEN THE LAST TWO WORKING
GROUPS WERE REALLY, YOU KNOW,
TRYING TO EMPHASIZE WHAT IS
IMPORTANT IS THAT WE PUT
PATIENTS FIRST AND HOW DO WE
OPTIMIZE AND HOW DO WE IMPROVE
THE QUALITY OF LIFE FOR A.L.S.
PATIENTS, AND WHAT DOES IT
REALLY MEAN, WHAT DOES IT MEAN,
QUALITY OF LIFE?
WHAT DO THE INDIVIDUALS LIVING
WITH THIS DISEASE ACTUALLY SEE
AS IMPORTANT FOR THEIR QUALITY
OF LIFE AND HOW CAN WE THEN MAKE
EFFORTS IN MAKING THAT HAPPEN
AND IMPROVING THOSE ASPECTS OF
IT?
AND THEN FINALLY WHAT IS REALLY
IMPORTANT, AND I THINK THIS HAS
COME UP THROUGHOUT THE MEETING
ACTUALLY TODAY, I HEARD THIS
HERE AND THERE, TO IMPROVE
COLLABORATIVE PARTNERSHIPS.
AND THOSE ARE REALLY IMPORTANT
IN REGARDS TO BRINGING TOGETHER
EVERYBODY THAT IS PART OF THIS
COMMUNITY.
STARTING FROM THE INDIVIDUALS
LIVING WITH THE DISEASE AND THE
CAREGIVERS TO THE RESEARCHERS
THAT SIT AT THE BENCH AND DO THE
RESEARCH TO THE CLINICIANS THAT
SEE PATIENTS AND RUN THE
CLINICAL TRIALS, BUT ALSO
THROUGH THE PHILANTHROPY THAT IS
THERE TO SUPPORT ALL OF THESE
EFFORTS AND REALLY BRINGING
EVERYBODY TOGETHER.
SO IF I CAN HAVE THE NEXT SLIDE,
I'M GOING TO PRESENT THE FIRST
THREE PRIORITIES THAT CAME OUT
OF THE WORKING GROUP OF THE
RESEARCH AND THE BIOLOGY BEHIND
A.L.S.
THIS WORKING GROUP WAS
CO-CHAIRED BY AMAR AL-CHALABI
AND CLOTILDE LAGIER-TOURENNE,
THERE'S A LOT OBVIOUSLY THAT
NEEDS TO BE DONE IN REGARDS TO
THE RESEARCH ON HOW WE BETTER
UNDERSTAND HOW THIS DISEASE IS
REALLY DEVELOPED.
AND THEY CAME UP WITH THREE
PRIORITIES WHICH SORT OF WERE
REALLY ACCEPTED ALSO BY THE
PUBLIC AND WERE COMMENTED ON BY
THE PUBLIC COMMENTS THAT WE GOT
LATER ON.
ONE IS TO REALLY FIGURE OUT WHAT
HAPPENS TO SPORADIC A.L.S.
MOST OF YOU KNOW THAT 10% OF
A.L.S. PATIENTS HAVE A FAMILY
HISTORY AND ARE CONSIDERED
FAMILIAL, BUT THE MAJORITY OF
PATIENTS, BETWEEN 85 AND 90, IS
REALLY SPORADIC, WHERE WE DON'T
HAVE A FAMILY HISTORY AND NOT
YET MANY GENES ARE ASSOCIATED
WITH THE DEVELOPMENT OF SPORADIC
A.L.S.
SO WE REALLY HAVE TO FIGURE OUT
MORE ABOUT HOW SPORADIC A.L.S.
MIGHT BE DEVELOPED IN THOSE
PATIENTS AND IN THAT PATIENT
POPULATION, AND HOW WE CAN
OBVIOUSLY THEN SUBSEQUENTLY
TARGET THIS.
THIS REALLY ALSO EMPHASIZES JUST
IN GENERAL THE DISEASE HETEROJE
NATE.
A.L.S. IS AN INCREDIBLE
HETEROGENEOUS DISEASE, NOT BASED
ON THE GENETICS AS I JUST
POINTED OUT BUT REALLY ALSO
BASED ON THE PATHOLOGY AND
DISEASE MECHANISMS ON THE ONSET
OF THE DISEASE.
EVERY PATIENT SORT OF HAS THEIR
OWN DEVELOPMENT AND PROGRESSION
OF THE DISEASE, AND THERE'S
REALLY A NEED FOR US TO BETTER
UNDERSTAND THIS AND TO THEN
AGAIN UTILIZE THIS KNOWLEDGE FOR
THE FUTURE DEVELOPMENT OF NOVEL
THERAPEUTICS.
AND THEN FINALLY, AND AGAIN THIS
SEEMS OBVIOUS, BUT IT WAS REALLY
AN EMPHASIS TO PUT FORWARD TO
REALLY HONE IN ON NOVEL
TECHNOLOGIES.
HOW CAN WE REACH OUT, HOW CAN WE
THINK OUTSIDE OF THE BOX, HOW
CAN WE INTEGRATE OTHER
DISCIPLINES THAT HELP US TO
REALLY STUDY THIS DISEASE IN
BETTER WAYS AND MORE EFFICIENT
WAYS AND FASTER WAYS.
IF I CAN HAVE THE NEXT SLIDE.
THIS WAS THE WORKING GROUP THAT
REALLY FOCUSED ON THE
TRANSLATIONAL ASPECTS OF THIS
DISEASE.
CO-CHAIRED BY DRS. NEIL
SCHNEIDER AND STACY WENINGER.
ONE WAS TO ESTABLISH WHAT THEY
CALLED A CENTERS OF EXCELLENCE.
WE CAN LATER DISCUSS WHETHER
THIS IS THE RIGHT NOMINATION OR
NOMENCLATURE THAT WE COULD USE,
BUT THE IDEA WAS TO REALLY HAVE
CENTERS THAT THEN COULD SORT OF
OVERSEE SOME OF THESE EFFORTS,
BUT REALLY ALSO INTEGRATE NOT
JUST, YOU KNOW, CENTERS THAT ARE
HOSTED IN BIG CITY, BUT REALLY
MAKE SURE THAT WE INTEGRATE ALSO
SMALLER CLINICS THAT ARE IN MORE
RURAL AREAS THAT REALLY NEED THE
KNOWLEDGE AND ALSO THE
INFRASTRUCTURE THAT ARE ALREADY
AVAILABLE IN THE LARGER CENTERS.
AND THAT ALSO LEADS TO ENHANCING
THE INFRASTRUCTURE NOT JUST FOR
PATIENT CARE AND ACCESS, BUT
REALLY ALSO FOR THINGS SUCH AS
BIOSAMPLE REPOSITORIES.
WE ALL KNOW THAT ONE WAY FOR US
TO BETTER UNDERSTAND AND TO
BETTER DIAGNOSE, TO FASTER
DIAGNOSE THE DISEASE ARE THINGS
LIKE BIOMARKER DEVELOPMENT.
BIOMARKERS CAN BE DEVELOPED WITH
THE RESOURCE OF VARYING
BIOSAMPLES THAT WE CAN HARVEST
AND THAT WE CAN OBTAIN FROM
PATIENTS.
CSF SAMPLES, BLOOD, SALIVA,
URINE.
ANY OF THESE SAMPLE REPOSITORIES
SHOULD REALLY SORT OF BE BETTER
ORGANIZED AND THERE SHOULD BE
ACCESS FOR EVERYBODY TO GET --
TO HAVE THE ABILITY TO TEST
THESE SAMPLES FOR SPECIFIC
HYPOTHESES WE HAVE TO DEVELOP
MORE STRONGER AND MORE RELIABLE
BIOMARKERS THAT CAN HELP US,
AGAIN WITH BETTER UNDERSTANDING
THE DISEASE AND POTENTIAL MAKING
BETTER DECISIONS ON CLINICAL
TRIAL DESIGN AND DEVELOPMENT AND
THEN FINALLY, REALLY ALSO IN THE
TRANSLATIONAL ASPECT, WE NEED
VERY EARLY ON TO GET EVERYBODY
TOGETHER AROUND THE TABLE.
INDUSTRY, ACADEMIA, WE HAVE TO
MAKE SURE THAT THIS WORKS
EASIER, THERE'S LESS BARRIERS,
LESS RED FLAGS.
IT IS SOMETIMES REALLY DIFFICULT
IN ORDER TO GET MTAs CROSSED
FROM ACADEMIA TO AN INDUSTRY,
WORKING TOGETHER WITH INDUSTRY
CAN SOMETIMES BE REALLY
DIFFICULT, AND THERE HAS TO BE A
WAY THAT WE FACILITATE THIS
INTERACTION BETWEEN EVERYBODY
WHO REALLY ONLY HAS A VERY
COMMON GOAL WHICH IS TO MOVE
FAST FORWARD TOWARDS A
DEVELOPMENT OF NOVEL
THERAPEUTICS.
SO I'M REQUESTIN GOING TO HAND R
TO MY CO-CHAIR BOB BROWN WHO
WILL INTRODUCE THE OTHER THREE
WORKING GROUPS OF OUR EFFORT.
>>YOU'RE MUTED, BOB!
>>THANK YOU.
SO THE THIRD GROUP WAS CHARGED
WITH THINKING ABOUT THE MOVEMENT
EFFORTS TO OPTIMIZE CLINICAL
RESEARCH AS WAS SHARED BY --
THIS WAS CHAIRED BY MERIT
CUDKOWICZ AND TIM MILLER.
FINALLY ENDORSED, ONE WAS THAT
IT WOULD LIKELY BE USEFUL IF
DONE PROPERLY TO HAVE A NEW
NATURAL HISTORY STUDY OF A.L.S.
WITH A PARTICULAR FOCUS ON THE
EARLIEST ASPECTS OF THE DISEASE.
AND TO THE EXTENT THAT ONE CAN,
PARTICULARLY IN A GENETIC
CONTEXT, LOOKING AT
PRESYMPTOMATIC ASPECTS OF THE
DISEASE.
THE THOUGHT WAS IF THIS WAS DONE
PROPERLY, IT WOULD BE
HYPOTHESIS-ENABLING AND IT WOULD
ULTIMATELY BE INCLUSIVE ENOUGH
TO ADDRESS SOME OF THE ISSUES
THAT WE'VE DISCUSSED PREVIOUSLY,
SUCH AS INCORPORATING A DIVERSE
SET OF PATIENTS.
BROADENING OUR PICTURE OF THE
SPECTRUM OF WHAT A.L.S.
PHENOTYPES CAN BE.
SO ONE OF THE ISSUES, THEREFORE,
WAS EXACTLY THAT, TO LOOK AT THE
BROADEST SET OF PATIENTS
POSSIBLE.
RIGHT NOW THERE IS A LIMITATION
IN TERMS OF UNDERREPRESENTED
MINORITIES AND ONE WOULD LIKE TO
DEAL WITH THAT SO THAT WE HAVE A
MUCH BROADER VIEW AGAIN OF THE
FULL COLORING OF THE SPECTRUM OF
A.L.S.
ONE OF THE ISSUES THAT WAS
ADDRESSED WAS THE NEED TO HAVE
BETTER BIOMARKERS THAT REFLECTED
BOTH PHARMACODYNAMICS OF
SPECIFIC DRUGS AS WELL AS THE
ACTUAL ACTIVITY OF THE DISEASE
ITSELF.
THE ISSUE OF OUT COME MEASURES
WAS DISCUSSED IN DETAIL.
YOU KNOW, FOR EXAMPLE, THE
PRESENTLY USED MOST COMMON
MARKER IS THE A.L.S. FUNCTIONAL
RATING SCALE WHICH IN FACT IS
ACCEPTED BY THE FDA, BUT IT HAS
SEVERAL LIMITATION.
IT ONLY APPLIES TO INDIVIDUALS
WHO ACTUALLY HAVE A.L.S., AND
HAS SEVERE LIMB TAIGS WHEN THERE
ARE BEHAVIORAL ASPECTS.
IT FOCUSES REALLY ONLY ON MOTOR
MANIFESTATIONS OF THE DISEASE
AND DOES NOT DISTINGUISH BETWEEN
PROGRESSION IN THE CNS AND
SPINAL CORD.
BIOMARKERS AND OUTCOMES VERY
SENSITIVE TO PRO IMRETION IN THE
EARLIEST AND THE LATEST STAGES
OF THE DISEASE.
ALSO ADDRESSED WITHIN THIS REALM
WAS THE IMPORTANCE OF NEW TYPES
OF CLINICAL TRIAL DESIGN SUCH AS
PLATFORM TRIALS.
AND THE THIRD COMPONENT OF THIS
WAS DEFINING THE EARLIEST
MANIFESTATIONS OF A.L.S. AS YOU
SEE HERE.
OBVIOUSLY THIS IS SOMETHING ONE
CAN PERHAPS DO MOST EASILY IN
THE CONTEXT OF MONOGENIC
CARRIERS OF THE DISEASE.
OR THOSE EVEN WITH A FAMILY
HISTORY WITHOUT A GENETIC CAUSE.
THIS IS MOST CHALLENGING FOR
SPORADIC A.L.S. CASES, IN WHICH
THE INDIVIDUAL MIGHT BE AT RISK
OF BECAUSE OF ENVIRONMENTAL
FACTORS, LIFESTYLE OR RATHER
CLINICAL EXPOSURES.
SO THIS WAS THOUGHT TO BE
ANOTHER VERY, VERY IMPORTANT
ASPECT OF THIS.
SO CONSIDER EFFORT WAS PUT TO
THE QUESTION ADDRESSING QUALITY
OF LIFE FOR PERSONS LIVING WITH
A.L.S.
THIS WAS A SESSION CHAIRED
BITTERY HEIMAN-PATTERSON AND
JEAN SWIDLER.
THIS FOCUSED ON THREE
PRIORITIES, IMPROVING AND
OPTIMIZING SYMPTOM MANAGEMENT,
OPTIMIZING FUNCTION AND
IDENTIFYING RESOURCES FOR A.L.S.
CARE.
THE FINAL WORKING GROUP
ADDRESSED THE IMPORTANT QUESTION
OF HOW TO GO BEYOND ANY ONE LAB
OR ANY ONE CENTER TOWARD
MEANINGFUL COLLABORATIONS AND
PARTNERSHIPS BOTH WITHIN
ACADEMIA AND POTENTIALLY WITH
ACADEMIA AND INDUSTRY.
THIS WAS CHAIRED BY DAN
DOCTOROFF AND LISA GOLD.
DR. DOCTOROFF HAS OF COURSE DONE
PIONEERING WORK IN THIS THROUGH
HIS FOUNDING A DIRECTORSHIP OF
TARGETING A.L.S.
WHICH INCLUDED NOT JUST DATA
ABOUT PATIENTS, PHENOTYPE,
BIOMARKERS AND SO FORTH BUT ALSO
SHARING INFORMATION ABOUT TOOLS,
ABOUT REAGENTS, ABOUT ALL OF
THOSE ELEMENTS THAT ARE
NECESSARY TO CONDUCT MEANINGFUL
RESEARCH, THAT WOULD BE THE
SECOND PRIORITY AS YOU CAN SEE
HERE.
AND THEN THE THIRD IS TO
ESTABLISH CLEARLY A BETTER WAY
TO OPTIMIZE COLLABORATIONS
BETWEEN ACADEMIA AND INDUSTRY,
RECOGNIZING AND WE HEARD THIS
DISCUSSED ACTUALLY IN THE
EARLIER SESSION THIS AFTERNOON,
THAT WHEN IT COMES TO
COMMERCIALIZATION AND WIDESPREAD
APPLICATION OF SOME DIAGNOSTIC
STUDIES AND THERAPEUTICS, THE
ASSOCIATION WITH INDUSTRY IS
INVALUABLE.
DR. DOCTOROFF WILL TALK A LITTLE
LATER AND HE MAY SAY MORE ABOUT
THIS.
SO I'LL TURN THE MICROPHONE BACK
TO RUTH.
RITA.>>THANKS, BOB.
I THINK ANOTHER ASPECT THAT WAS
REALLY IMPORTANT IN THIS EFFORT
WAS THAT WE HAD, AT THE PUBLIC
WORKSHOP THAT WE HELD IN
OCTOBER, THAT WE INTEGRATED NOT
JUST A DISCUSSION ABOUT THE
DIFFERENT WORKING GROUPS AND THE
PRIORITIES THAT WERE SET BY THE
WORKING GROUPS, THAT WE ALSO
INTEGRATED A SESSION WE CALLED A
LIVED EXPERIENCE PANEL.
THAT WAS LED BY LAYNE AND ELLEN
PHILLIPS.
THEY WILL GIVE YOU SOME COMMENTS
ON THEIR EXPERIENCE WITH WHAT
THEY DID DURING THIS EFFORT
DURING THE WORKSHOP AND HOW THEY
FELT THIS GREATLY CONTRIBUTED TO
THE OVERALL CONCEPT AND THE
PUTTING TOGETHER OF THIS WORKING
PLAN.
SO I JUST WANT TO EMPHASIZE
THAT.
I THINK WE CAN SKIP THAT SLIDE
BECAUSE WHAT I WOULD REALLY --
CAN WE GO TO THE NEXT ONE?
THIS IS REALLY JUST A SUMMARY OF
WHAT WE JUST HEARD, BUT WHAT WE
WANTED TO REALLY POINT OUT IS
THAT WHEN WE POSTED THIS PLAN
BACK TO THE PUBLIC AFTER WE HAD
THE WORKSHOP, WE GOT MORE
COMMENTS BACK, AND SO WHAT
REALLY, YOU KNOW, SORT OF CAME
ACROSS FROM ALL OF THE COMMENTS
THAT WE RECEIVED FOR THE SECOND
TIME, SO THE PUBLIC HAD REALLY A
LOT OF OPPORTUNITIES TO
CONTRIBUTE TO THIS, WAS THAT
REALLY THE URGENCY LIES IN
FIGURING OUT WHAT HAPPENS
PRESYMPTOMATICALLY AND HOW CAN
WE IMPROVE ON BIOMARKER
DEVELOPMENT.
SO THAT WAS FELT TO BE REALLY AN
IMPORTANT ASPECT AND WE SORT
OF -- WE WANTED TO MAYBE TRY AND
GIVE PRIORITIES TO THE
PRIORITIES BECAUSE WE OBVIOUSLY
REALIZE THAT 15 PRIORITIES IS A
LOT TO HANDLE, AND SO WE'RE
TRYING TO LISTEN TO THE PUBLIC,
WHERE THEY FELT AMONG THOSE 15
PRIORITIES WHERE SHOULD WE
REALLY START.
MAYBE THERE ARE SOME LOW HANGING
FRUIT THAT WE CAN TACKLE FIRST,
BEFORE WE GO TO THE MORE COMPLEX
ISSUES THAT WERE BROUGHT UP THAT
ARE YET, YOU KNOW, NEVERTHELESS
EQUALLY IMPORTANT TO THIS
COMMUNITY.
THE SECOND PART WAS REALLY THE
INCLUSION OF INDIVIDUALS AT RISK
FOR A.L.S. AND HOW CAN WE
INCLUDE THESE INDIVIDUALS IN
CLINICAL TRIAL DESIGNS.
EVERY MEETING THAT I'VE ATTENDED
OVER THE LAST YEAR BROUGHT THIS
UP AS A VERY CRITICAL DISCUSSION
POINT ON HOW CAN WE INTEGRATE
INDIVIDUAL THAT ARE AT RISK INTO
CLINICAL TRIALS THAT WE RUN THAT
USUALLY ARE RUN ONLY FOR
INDIVIDUALS THAT ALREADY HAVE
SYMPTOMS, AND HOW CAN WE SAFELY
INTEGRATE THESE INDIVIDUALS INTO
THOSE CLINICAL TRIAL DESIGNS TO
GIVE THEM AN OPPORTUNITY TO BE
PART OF THIS BECAUSE WE ALL KNOW
THE SAYING IS, THE EARLIER WE GO
IN, THE HIGHER LIKELIHOOD WE'RE
GOING TO HAVE IN ORDER TO SEE
EFFICACY WITH ANY OF THE
THERAPEUTICS THAT WE'RE TRIALING
FOR THESE PATIENTS AND FOR THIS
PATIENT POPULATION.
AND SOMETHING THAT BOB HAD
BROUGHT UP DURING THE DISCUSSION
ALREADY IN SOME OF THE
PRIORITIES, DIVERSITY AND
INCLUSIVITY.
THERE WAS REALLY A BIG EFFORT
FROM THE PUBLIC TO POINT OUT
THAT WE NEED TO MAKE SURE THAT
WE INTEGRATE INTO THE CENTERS OF
EXCELLENCE, MAYBE WE CAN CALL
THEM COOPERATIVE GROUPS, TO
REALLY MAKE SURE THAT WE
INTEGRATE EVERYBODY.
WE GO TO RURAL AREAS, WE
INTEGRATE MINORITIES AND DIVERSE
POPULATIONS INTO THE CLINICAL
TRIAL DESIGN, THE CARE DESIGNS
WE DO, THE RESEARCH TRIALS WE
DO, NOT JUST THE THERAPEUTIC
TRIALS BUT ALSO THE RESEARCH
TRIALS FOR THE COLLECTION OF
BIOMARKERS AND ALL OF THESE
EFFORTS.
SO THIS WAS REALLY A BIG
IMPORTANT ASPECT THAT WE WANTED
TO HIGHLIGHT AMONG THOSE
PRIORITIES THAT WE JUST
PRESENTED.
AND SO IF I CAN HAVE THE NEXT
SLIDE, THIS ONE, I TITLED THE
CONCERNS, THE RECURRING CONCERNS
THAT CAME FROM THE PUBLIC, AND I
JUST WANT TO REITERATE THOSE.
THESE CAME ALSO FROM DISCUSSIONS
THAT I HAD AFTER TALKING TO A
LOT OF INDIVIDUALS THAT ARE SORT
OF PART OF THIS COMMUNITY, AND
ONE OF THE IMPORTANT ASPECTS
CAME BACK OVER AND OVER AGAIN
AND THAT IS THE A.L.S. CLOCK AS
THE COMMUNITY REALLY CALLS IT.
AND THAT IS THE URGENCY THAT
THESE INDIVIDUALS HAVE A VERY
SHORT LIFESPAN.
WE ALL KNOW THAT THE MEDIAN
LIFESPAN OF AN A.L.S. PATIENT
AFTER BEING DIAGNOSED IS THREE
TO FIVE YEARS.
SO THE URGENCY OF REALLY MOVING
FORWARD AND IN ADDRESSING THESE
PRIORITIES IS THERE, AND THIS
CAME UP OVER AND OVER AGAIN, AND
WE REALLY WANTED TO MAKE SURE
THAT THE COUNCIL LISTENS TO THE
COMMENTS OF THE PUBLIC AND THE
CONCERNS THAT WAITING FOR A YEAR
IS A LONG TIME FOR THIS
POPULATION, FOR THIS PATIENT
POPULATION, AND THEY'RE REALLY
HOPING TO SEE THAT ACTION WILL
BE THERE IN A SHORTER PERIOD OF
TIME.
AND THAT SORT OF LEADS INTO THE
SECOND POINT IN THE
IMPLEMENTATION OF THE STRATEGIC
PLAN.
HOW ARE WE GOING TO IMPLEMENT
THESE PRIORITIES?
SHORT TERM, AS I SAID, LOW
HANGING FRUIT, WHAT CAN BE DONE
FAIRLY QUICKLY AND MAYBE LONG
TERM ON HOW WE ADDRESS THIS.
AND THEN WITH THIS FOLLOWING,
HOW DO WE MONITOR THE PROGRESS
ON THESE SET PRIORITIES?
IS THERE A WAY THAT WE CAN SET
SOMETHING IN PLACE THAT WE SORT
OF HAVE CHECKS AND BALANCES AND,
YOU KNOW, LOOKING BACK, HAVE WE
ADDRESSED ANY OF THESE ISSUES OR
ARE WE JUST GOING TO SIT THERE
AND WAIT UNTIL SOMETHING HAPPENS
WITHOUT ANY ACTION AND ANY
ACTIVITY AND ANY FEEDBACK TO THE
COMMUNITY TO SAY, LOOK, WE'VE
REALLY MADE AN EFFORT AND WE'VE
MANAGED TO ADDRESS PRIORITY A, B
AND C.
SO THIS IS SOMETHING THAT WE
WOULD LIKE TO PUT OUT FOR
DISCUSSION AND WE WOULD REALLY
APPRECIATE ANY FEEDBACK THAT WE
CAN GET FROM THE COUNCIL ON
THESE ASPECTS THAT WE' WERE REAY
RAISED AS IMPORTANT ISSUES BY
THE COMMUNITY WHEN WE PROVIDED
OUR STRATEGIC PLAN TO THE
PUBLIC.
SO WITH THAT, WE WOULD LIKE TO
INTRODUCE THE NEXT THREE
SPEAKERS, AND BOB IS ACTUALLY
GOING TO DO THAT.
SO I HAND IT OVER TO BOB FROM
HERE.
>>I'LL BE BRIEF.
YOU'VE ALREADY BEEN INTRODUCED
TO THEM.
ONE IS ELL ELLYN PHILLIPS, THE R
OF THE GREATER PHILADELPHIA
CHAPTER, SHE/HER SELF WAS AN
A.L.S. CAREGIVER.
THE SECOND IS LAYNE OLIFF,
RESEARCH AMBASSADOR FOR THE
NORTHEAST A.L.S. CONSORTIUM, AND
HE'S ALSO A FELLOW AND ACTIVE IN
THE INTERNATIONAL ALLIANCE OF
A.L.S./MND.
THE THIRD IS OUR COLLEAGUE AND
FRIEND DAN DOCTOROFF, WHO'S
ALREADY BEEN MENTIONED, WHO
HIMSELF HAS BEEN CONTENDING WITH
A.L.S.
HE'S THE FORMER CHAIR AS YOU'VE
HEARD AND CEO OF SIDEWALK LABS.
HE HAS HAD AN IMMENSE IMPACT ON
THE FIELD THROUGH THE CREATION
OF TARGET A.L.S., WHICH REALLY
HAS BROUGHT TOGETHER MANY
ACADEMIC LABS AND COMPANIES,
LARGE AND SMALL, AND FORMER
PRESIDENT AND CEO OF BLOOK BERG
PARTNERS, ALSO DEPUTY MAYOR OF
NEW YORK CITY IN CHARGE OF
ECONOMIC DEVELOPMENT.
IT'S BEEN FANTASTIC HAVING HIM
ON THIS TEAM.
SO WHY DON'T WE GO NOW TO ELLYN
AND LAYNE.
>>THANK YOU, BOB.
CAN YOU PUT THE SLIDES UP,
PLEASE?
THANK YOU.
ELLYN AND I WILL TAKE THIS
SECTION.
LET ME INTRODUCE MYSELF, LAYNE
OLIFF.
AS YOU CAN TELL BY THE WAY I
TALK, I HAVE A.L.S.
I AM A PHARMACIST BY TRAINING.
I WAS ORIGINALLY DIAGNOSED WITH
PLS IN 2017.
THAT WAS CHANGED TO A.L.S. ON
NOVEMBER 11TH OF 2020.
ENOUGH OF MY BACKGROUND.
I'D LIKE TO THANK FIRST THE
WORKING GROUPS, DR. SATTLER, DR,
DR. TORBORG AND ESPECIALLY ELLYN
PHILLIPS.
ELLYN AND I SPENT MANY HOURS
ACTUALLY TALKING ABOUT OUR ROLE
IN THE STEERING COMMITTEE AND
WORKING WITH THE WORKING GROUPS.
SO WORKING WITH ELLYN WAS A
PLEASURE.
I'LL TURN IT OVER TO EL IN ELLYW
TO TAKE OVER THE NEXT COUPLE
SLIDES.
ELLYN?
>>THANK YOU, LAYNE.
I FEEL THE SAME WAY ABOUT YOU.
I THINK YOU KNOW THAT.
IT'S GREAT TO BE BACK AT
COUNCIL, WHERE I SERVED FROM
2001 TO 2005.
I FIRST LEARNED ABOUT A.L.S.
WHEN MY HUSBAND ALLAN WAS
DIAGNOSED IN 1982 AT THE AGE OF
34.
DESPITE EXTRAORDINARY CARE AND
EMPATHY BY DR. FISHBACH, TWO
YEARS LATER, WHEN OUR PRECIOUS
DAUGHTER AMY WAS 3, HE DIED.
OUR WORLD WAS TURNED
UPSIDE-DOWN.
I'M SURE SO MANY OF YOU KNOW HOW
THAT FEELS.
FOR 40 YEARS, I HAVE HOPED,
PRAYED, AND WORKED TO HELP FIND
AN ANSWER TO A.L.S., AND I KNOW
I HAVEN'T BEEN DOING THIS ALONE.
IT WAS JANUARY 30TH, A YEAR AGO,
THAT DR. KOROSHETZ EMAILED ME
WITH AN INVITATION TO JOIN THE
STEERING COMMITTEE OF THE FIRST
EVER A.L.S. STRATEGIC PLAN.
I COULDN'T SAY NO, EVEN THOUGH I
HAD NO IDEA WHAT I WAS GETTING
MYSELF INTO.
THIS LOYAL GROUP WHO I GREW UP
WITH, SO MANY OF YOU, YOUR
DEDICATION HAS NEVER WANED.
I MET EXTRAORDINARY PEOPLE LIKE
JEAN SWITLER.
I WOULD ALWAYS WANT HER ON MY
TEAM.
AND I'VE ALREADY SPOKEN ABOUT
LAYNE.
HE'S THOUGHTFUL, INTELLIGENT,
AND HAS HELPED ME NAVIGATE
THROUGH THIS STRATEGIC PLAN
PROCESS.
INTERACTING WITH THE TIRELESS
MEMBERS OF NIH AND NINDS
PROVIDED INSIGHT INTO HOW
IMPORTANT THEIR CONNECTION IS TO
THE SUCCESS OF THE WORK WE HAVE
JUST COMPLETED.
COULD I HAVE SLIDE 2, PLEASE?
WHEN YOU LOOK AT THIS SLIDE
BELOW THE LIST OF THE STEERING
COMMITTEE, SORRY, AND WORKING
GROUP -- CAN WE SCROLL DOWN A
LITTLE BIT ON THAT SLIDE?
SORRY.
>>GO BACK TWO SLIDES.
>>SORRY, GO BACK TWO SLIDES.
YES, I'M SORRY.
WE SEE AT THE BOTTOM OF THIS
SLIDE, AND THIS IS SO CRUCIAL,
11 OF 52 PARTICIPANTS ARE "ALS
EXPERIENCED EXPERTS."
AND ON SLIDE 3, PLEASE GO TO
SLIDE 3, THE IMPORTANCE OF
A.L.S. EXPERIENCED EXPERTS
BECOMES OBVIOUS.
EVERY WORKING GROUP HAD A PALS,
CALS OR GENE CARRIER.
THIS IS THE FIRST TIME THIS HAS
BEEN DONE, AND HOPEFULLY IT
WON'T BE THE LAST.
I WANT TO THANK YOU,
DR. KOROSHETZ, AND ALL THE
MEMBERS OF THE STRATEGIC
PLANNING COMMITTEE WHO HAVE
GIVEN HOURS AND HOURS OF WORK TO
PRODUCE THIS DOCUMENT, WHICH
WILL BE USED TO MOVE OUR EFFORTS
AHEAD SO THAT SOME DAY,
HOPEFULLY SOON, A 3-YEAR-OLD
WILL NOT LOSE HER FATHER TO
A.L.S., AND EFFECTIVE TREATMENTS
WILL BE AVAILABLE TO ALL TO END
THE SCOURGE THAT IS A.L.S.
THANK YOU.
>>CAN I HAVE THE NEXT SLIDE,
PLEASE.
SO, ELLYN TALKED ABOUT THE ROLE
OF THE A.L.S. EXPERIENCE
EXPERTS, SUCH AS MYSELF.
WE WERE INVOLVED IN ALL STAGES
OF THE DEVELOPMENT OF THE
STRATEGIC PLAN, FROM INITIATION
TO BEING HERE TODAY, TO ACTUALLY
PRESENT TO YOU.
WE WERE INTIMATELY INVOLVED IN
THE DOCUMENT YOU RECEIVED, WHICH
IS A PROPOSED FINAL DRAFT PLAN,
SO WE WERE INVOLVED WITH ALL
ASPECTS IN DEVELOPMENT OF THIS
DOCUMENT.
AND MORE IMPORTANT IS, WE WERE
WELL RECEIVED.
I TALKED TO ALL OF THE A.L.S.
EXPERIENCE EXPERTS INVOLVED WITH
THE WORKING GROUPS AND THE
STEERING COMMITTEE.
ALL WERE WELL RECEIVED.
THEY LISTENED, THEY DISCUSSED,
AND THEY ADDED VALUABLE INPUT TO
THE PROCESS, AND AS ELLYN HAS
STATED BEFORE, WE DID WORK
INVOLVED WITH ALL WORKING GROUP
MEETINGS.
MOST MEETINGS WERE HELD
VIRTUALLY, AND WE PARTICIPATED
IN ALL OF THEM.
NEXT SLIDE, PLEASE.
SO, WHERE DO WE GO FROM HERE?
TALKING TO THE PEOPLE IN THE
A.L.S. COMMUNITY, DR. SATTLER
TOUCHED ON THIS ALREADY, WE ARE
PROPOSING SEVERAL ELEMENTS UPON
APPROVAL OF THE STRATEGIC PLAN.
WE NEED TO FIRST DISSEMINATE,
EDUCATE, AND PROMOTE THIS PLAN
FOR EVERYBODY IN THE A.L.S.
COMMUNITY, NOT JUST PATIENTS,
NOT JUST RESEARCHERS, NOT JUST
CLINICIANS.
WE NEED TO EDUCATE EVERYBODY IN
THE COMMUNITY.
RAISE AWARENESS, AS I WOULD SAY.
NUMBER ONE.
NUMBER TWO, DR. SATTLER
MENTIONED THIS ABOUT AN
IMPLEMENTATION WORKING GROUP TO
MONITOR THE SUCCESS, CHALLENGES
AND OPPORTUNITIES WITHIN THE
STRATEGIC PLAN TO DETERMINE IF
THE GOALS ARE GOING TO BE MET OR
IF GOALS ARE ACTUALLY MET OR IF
WE'RE A LONG WAY TO MEETING
CERTAIN GOALS.
AND LAST BUT NOT LEAST, MANY OF
YOU AT THIS MEETING KNOW THAT
A.L.S. EXPERIENCE EXPERTS ARE
THE EYES AND EARS, SO TO SPEAK,
OF THE A.L.S. COMMUNITY.
WE HEAR MUCH OF WHAT'S GOING ON
OUT THERE, AND WE CAN DETERMINE
HOW THE PLAN NEEDS TO BE
ADAPTED, CHANGED OR MADE
STREAMLINED BASED ON CHALLENGES
AND OPPORTUNITIES IN THE
CHANGING ENVIRONMENT.
SO WE CAN HELP THIS PROCESS MOVE
ALONG.
OVERALL, IT WAS A GREAT
EXPERIENCE.
LAST SLIDE, PLEASE.
MOST OF YOU KNOW THIS IS A
COMMENT OR A STATEMENT FROM
MICHAEL J. FOX THAT'S FOCUSED ON
PARKINSON'S DISEASE, BUT IT'S
VERY RELEVANT TO THE A.L.S.
COMMUNITY.
THERE ARE MANY, MANY
RESEARCHERS, CLINICIANS,
ACADEMICIAN EXPERTS OUT THERE
WHO ARE EXPERTS IN A.L.S.
IN ADDITION, WE REALLY WANT TO
THANK NIH, AND NINDS FOR RAISING
AWARENESS OF THE ROLE OF A.L.S.
EXPERIENCE EXPERTS, PALS, CALS,
GENE CARRIERS.
WE ARE EXPERTS ALSO, AND
LISTENING TO US HAS MADE THIS
STRATEGIC PLAN EVEN MORE
VALUABLE TO THE A.L.S.
COMMUNITY.
SO THANK YOU VERY MUCH, NIH AND
NINDS, FOR ALL YOU'VE DONE TO
RAISE AWARENESS OF A.L.S. AND
UTILIZE PEOPLE WHO ARE AFFECTED
BY THIS AWFUL DISEASE.
THANK YOU.
DAN, I'LL TURN IT OVER TO YOU.
>>YOU'D THINK BY NOW I'D LEARN
TO UNMUTE MYSELF.
THANK YOU VERY MUCH, LAYNE AND
ELLYN.
AND RITA AND BOB AND AMELIE.
I THOUGHT MAYBE I WOULD JUST
GIVE A LITTLE BIT OF BACKGROUND
ON MY CONNECTION TO A.L.S.
MY FATHER AND UNCLE BOTH DIED OF
A.L.S.
MY UNCLE IN 2010.
AND AFTER MY UNCLE DIED, WHICH
WAS AFTER MY FATHER DIED, I
DECIDED THAT I HAD TO DO
SOMETHING.
BECAUSE CLEARLY, IT WAS
HEREDITARY IN MY FAMILY.
THEY ALSO, BY THE WAY, HAD A
FIRST COUSIN WHO DIED OF FTE,
THE SAME GENETIC MUTATION.
SO AT THAT POINT IN 2011, 2012,
I CREATED TARGET A.L.S.
AND TARGET A.L.S.' MISSION, AND
I'LL BE VERY BRIEF, IS TO
ACCELERATE RESEARCH INTO
CLINICAL TRIALS.
EXCLUSIVELY RESEARCH-BASED.
THE WAY WE GO ABOUT IT IS IN A
SERIES OF INITIATIVES.
ONE IS WE ONLY FUND
MULTIDISCIPLINARY CONSORTIA OF
RESEARCHERS, OFTEN LED BY
INDUSTRY INVESTIGATORS, AND SO
THE COMBINATION OF INDUSTRY AND
ACADEMIA, WE THINK IS VERY
POWERFUL.
SECONDLY WHAT WE DO IS WE FUND A
SUITE OF CORE SCIENTIFIC
RESOURCES THAT ANY RESEARCHER
AROUND THE WORLD CAN DRAW ON.
AND THOSE INCLUDE ANTIGENS,
LONGITUDINAL BIOFLUIDS,
POSTMORTEM TISSUE SAMPLES, STEM
CELLS, ANIMAL MODELS, VIRAL
VECTORS, AND THE LIKE.
THOSE THINGS HAVE BEEN ACCESSED
ON SOMETHING LIKE A THOUSAND
DIFFERENT PROJECTS OVER THE LAST
SIX OR SEVEN YEARS.
THIRD, WE FOCUS ON ENGAGING
INDUSTRY IN EVERYTHING THAT WE
DO.
AS I MENTIONED, THEY'VE LED
ABOUT 50% OF OUR CONSORTIA.
WE'VE HAD FUNDED 53 DIFFERENT
CONSORTIA, OF WHICH, BY THE WAY,
60% HAVE LED TO CONTINUING DRUG
DISCOVERY PROGRAMS AT BIOTECH OR
PHARMA COMPANIES.
AND HAVE LED TO
SIX CLINICAL TRIALS.
WE ALSO HAVE THEM ON OUR
INDEPENDENT REVIEW COMMITTEE,
ABOUT 50%, AND ROUGHLY 50% OF
OUR BOARD.
WE ARE CURRENTLY ENGAGED WITH,
ON AN ACTIVE BASIS, 123
DIFFERENT BIOTECH, PHARMA, AND
BC FIRMS, WHICH IS A REAL
TESTAMENT TO THE PROGRESS THAT
HAS BEEN MADE.
THE FACT THAT WE BRING PEOPLE
TOGETHER HAS GIVEN US THE
CREDIBILITY LEAD INDUSTRY-WIDE
INITIATIVES LIKE THE SEARCH FOR
A BIOMARKER.
AND SO WE BELIEVE WE'VE
DEVELOPED A SUCCESSFUL MODEL.
NOW, AS BOB MENTIONED, BOB BROWN
MENTIONED, IN LATE 2021, I WAS
DIAGNOSED WITH A.L.S.
WEIRDLY, AND BOB CAN'T EXPLAIN
THIS, I DO NOT HAVE THE SAME
GENETIC MUTATION AS MY UNCLE,
FATHER, AND THEIR FIRST COUSIN.
I DON'T HAVE ONE THAT HAS BEEN
IDENTIFIED YET.
BUT WHEN I WAS DIAGNOSED, THEY
SAID, YOU KNOW, MY LAST MISSION
IS GOING TO BE TO DRAMATICALLY
SCALE UP TARGET A.L.S.
AND I SET A GOAL OF RAISING
$250 MILLION, AND I WILL JUST
SAY I'M WELL ON THE WAY TO
ACHIEVING THAT GOAL.
LOOK, I WAS REALLY HONORED TO BE
PART OF THE NIH NINDS STRATEGIC
PLANNING PROCESS, AND I'VE BEEN
ASKED JUST TO COMMENT ON THAT
PROCESS, AND I THINK WHERE WE GO
FROM HERE.
YOU KNOW, I THINK MOST OF YOU
KNOW THAT OVER THE LAST 10
YEARS, THERE HAS BEEN REALLY
REMARKABLE PROGRESS MADE IN
A.L.S. RESEARCH.
WE UNDERSTAND DRAMATICALLY
BETTER THE BIOLOGY OF THE
DISEASE, THE GENETICS OF THE
DISEASE.
WE HAVE DRAWN IN HUNDREDS OF NEW
INVESTIGATORS, BOTH IN INDUSTRY
AND IN ACADEMIA.
WE HAVE ENGAGEMENT BY BIOTECH
AND PHARMA AND VC, I THINK HAS
SURPASSED EVERYONE'S
EXPECTATIONS, AND WE ARE MOVING
FROM RESEARCH INTO THE CLINIC.
AND SO THE PIECES ARE BEING PUT
TOGETHER TO ABSOLUTELY BE ABLE
TO FIND TREATMENTS FOR PEOPLE.
WE'RE NOT THERE YET.
BUT WE ARE GETTING CLOSER AND
CLOSER.
A BIG PART OF THE SUCCESS THAT
WE HAVE SEEN OVER THE LAST
DECADE HAS BEEN FROM NIH AND
NINDS.
YOU KNOW, THE TOTAL FUNDING OVER
THAT PAST DECADE FROM I THINK
2014 TO 2022 WENT FROM
$48 MILLION TO ABOUT
$124 MILLION.
THERE'S BEEN INCREASED
PHILANTHROPY, INCLUDING THE ICE
BUCKET CHALLENGE AND OTHER
ORGANIZATIONS.
AND THERE IS AN INCREASING
AMOUNT OF HOPE.
BUT I THINK WHAT IS MOST
IMPORTANT IS THAT THERE'S BEEN
REMARKABLE COLLABORATION AMONG
ALL OF THE CONSTITUENTS IN
A.L.S. RESEARCH.
AND THAT INCLUDES OBVIOUSLY
PATIENTS, CLINICIANS, ACADEMICS,
INDUSTRY, NON-PROFIT, AND, OF
COURSE, GOVERNMENT, WHO HAVE
BEEN WORKING TOGETHER WITH A --
I WON'T SAY A COMPLETELY COMMON
PURPOSE, BUT WITH A SENSE OF
HOMOMENTUM AND THE FEELING THATF
WE WORK TOGETHER, WE CAN
ACTUALLY BEGIN TO SOLVE THESE
PROBLEMS.
THE STRATEGIC PLANNING PROCESS
THAT HAS ALREADY BEEN DESCRIBED
FOR YOU OVER THIS LAST YEAR OR
SO, I THINK IS REALLY A
REFLECTION, IT'S MAYBE THE
APOTHEOSIS OF THAT SORT OF LEVEL
OF COLLABORATION.
BECAUSE ALL PARTIES ACTUALLY
WORK TOGETHER AS LAYNE AND ELLYN
HAVE DESCRIBED IN A WAY THAT I
DON'T THINK THEY EVER HAVE
BEFORE, TO ENSURE THAT WE HAVE A
NORTH STAR WITH THE GOAL
ULTIMATELY OF SAVING EVERYONE'S
LIFE.
AND THIS IS A DISEASE WHERE 1 IN
400 PEOPLE WILL DIE OF A.L.S.
UNLESS WE FIND TREATMENTS.
SO I THINK WHAT WE HAVE DONE
WITH AN UNUSUAL AMOUNT OF
CONSENSUS IS LAID OUT THAT PATH.
NOW IN MANY CASES OF THE 15
DIFFERENT INITIATIVES, WE ARE ON
THAT PATH, BUT THE QUESTION IS,
HOW DO WE ACCELERATE IT?
AND HAVING A CLEAR PLAN, WHICH
THE FIELD AGREES WITH, IS REALLY
CRITICAL TO ACHIEVING THAT GOAL.
NOW, THE QUESTION IS, HAVING
THIS ALIGNMENT, HOW DO WE
ACTUALLY OPERATIONALIZE THE PLAN
GOING FORWARD?
RIGHT?
AND, YOU KNOW, LOOK, FIRST OF
ALL, HAVING MORE MONEY WHICH THE
ACT FOR A.L.S. GIVES US THE
POTENTIAL TO DO, IS CERTAINLY AN
IMPORTANT PART OF THAT.
THE ACT FOR A.L.S. ALLOCATES UP
TO $100 MILLION A YEAR.
SOME OF THAT WILL CERTAINLY GO
TOTO, EXPANDED ACCESS PROGRAMS,
PROBABLY THE MAJORITY OVER TIME
WILL GO TO IT.
BUT THEN THERE WILL BE
SIGNIFICANT FUNDS LEFT OVER FOR
PARTNERSHIPS AND COLLABORATIONS
UNDER THE ACT, ARTICLE 3 AND
ARTICLE 5.
BUT WHAT I'VE SEEN,
INTERESTINGLY, AND THIS FOLLOWS
UP ON SOME OF THE COMMENTS THAT
LAYNE MADE, IS THAT NIH AND
NINDS, WITH THIS AGREEMENT ON
WHAT TO DO, HAVE BEEN ACTING
AFTER WE'VE DEVELOPED THE PLAN
WITH A SENSE OF URGENCY THAT I
THINK BEFITS THE NATURE OF THIS
DISEASE.
80% OF PATIENTS DIE WITHIN FIVE
YEARS.
AND NOW HAVING AGREED ON AN
APPROACH, A SET OF INITIATIVES,
A SET OF GOALS, I THINK WHAT I'M
OBSERVING IS THAT THERE IS A
INTENSE SENSE OF URGENCY THAT IS
BEING APPLIED TO AT LEAST
CERTAIN OF SOME OF THESE
INITIATIVES.
NOW, I SERVED IN GOVERNMENT, AS
BOB SAID.
I WAS RESPONSIBLE FOR THE
REBUILDING OF NEW YORK AFTER
9/11.
AND ONE THING I LEARNED ABOUT
GOVERNMENT IS THAT YOU HAVE TO
OPERATE IN GOVERNMENT ON TWO
SPEEDS AT THE SAME TIME.
YOU NEED TO BE PATIENT, BECAUSE
THERE ARE SO MANY PARTIES TO BE
TOUCHED AND INCLUDED AND IF YOU
NEED TO COLLABORATE AMONG THOSE,
IT CRITICAL THAT YOU BE PATIENT,
BUT AT THE SAME TIME, YOU HAVE
TO ACT WITH A SENSE OF URGENCY
AT ALL TIMES.
AND WHAT I AM SEEING OUT OF
NINDH, NINDS AND NIH, IS THAT
SENSE OF URGENCY, THAT THIS IS A
DISEASE AT THIS MOMENT THAT WE
CAN TACKLE WITH ENOUGH FOCUS,
ENOUGH RESOURCES, AND ENOUGH
COLLABORATION, AND THAT IS WHY I
AM ACTUALLY SO HOPEFUL.
SO AGAIN, I WAS TRULY HONORED TO
BE A PART OF THIS PROCESS, AND
EXCITED HOPEFULLY TO BE A PART
OF MAKING IT HAPPEN IN THE
FUTURE.
THANK YOU.
>>THANK YOU, DO DON, AND THANKU
FOR A REALLY EXCELLENT SYNOPSIS
OF THE PLAN AND ALSO YOUR
IMPORTANT PERSPECTIVE, AND OF
COURSE THE QUESTIONS, WHAT COMES
NEXT.
SO I THINK WE HAVE A LITTLE BIT
OF TIME FOR QUESTIONS, I KNOW WE
ALSO THEN HAVE TO INVITE OUR
NEXT SPEAKER BUT IF ANYONE HAS
QUESTIONS, PLEASE RAISE YOUR
HANDS, AND I THINK BOB YOU ALSO
NEED A VOTE, RIGHT?
>>YES.
I ACTUALLY DIDN'T REALIZE WE
NEED A VOTE BUT WE CAN HAVE THAT
WHEN WE'RE DONE.
>>WE'RE LOOKING FOR A VOTE TO
APPROVE THE STRATEGIC PLAN.
>>YES, SO WE'RE LOOKING FOR A
MOTION TO APPROVE THE STRATEGIC
PLAN RECOMMENDATIONS, THIS IS
GOVERNMENT-SPEAK.
DOESN'T MEAN YOU NECESSARILY
AGREE WITH EVERY SINGLE ONE BUT
IT MEANS THAT YOU'VE HEARD AND
RECEIVED THEM.
>>SO MOVED.
>>SECOND?
>>SECOND.
>>THEN WE CAN VOTE IN THE CHAT.
THANKS.
THANKS A LOT.
BACK TO YOU, AMELIE.
>>ALL RIGHT.
SO I'M GOING TO INTRODUCE OUR
NEXT SPEAKER AND THAT'S
DR. JACQUELINE CORRIGAN-CURAY,
PRINCIPAL DEPUTY CENTER
DIRECTOR, CENTER FOR DRUG
EVALUATION AND RESEARCH, FDA.
AND JACQUELINE IS GOING TO TALK
A LITTLE ABOUT FDA ACTIVITIES IN
THE CONTEXT OF ACT FOR A.L.S.
IMPLEMENTATIONS.
THANK YOU.
>>THANK YOU.
LET ME PULL UP MY SLIDES.
YOU SHOULD BE SEEING THEM ON THE
SCREEN RIGHT NOW.
FIRFIRST OF ALL CONGRATULATIONSN
THE STRATEGIC PLAN AND ALL THE
INPUT.
VERY IMPRESSIVE.
I'M GOING TO TURN AND TALK A
LITTLE BIT ABOUT WHAT OUR
ACTIVITIES ARE IN SUPPORT OF
IMPLEMENTING THE ACCELERATING
ACCESS TO CRITICAL THERAPIES FOR
A.L.S. ACT.
SO I'M GOING TO GIVE YOU A QUICK
OVERVIEW AND FOCUS ON WHERE
WE'RE RESPONSIBLE IN TERMS OF
THE GRANT PROGRAM, WORKING WITH
NIH ON THE PPP AS WELL AS THE
ACTION PLAN.
SO THIS IS JUST THE SECTION OF
THE ACT AS MANY OF YOU KNOW, NIH
TAKES A LEAD ON SECTION 2, THE
GRANTS FOR RESEARCH.
WE WERE ASKED TO COLLABORATE
WITH NIH IN THE PUBLIC-PRIVATE
PARTNERSHIP, AND THEN WE HAD OUR
OWN ACTIVITIES AROUND RARE
NEURODEGENERATIVE DISEASE ACTION
PLAN AND A GRANT PROGRAM, AND OF
COURSE THE ACT WILL BE EVALUATED
OR OUR PROGRESS UNDER IT WILL BE
EVALUATED BY THE GAO.
SO LET'S TALK A LITTLE BIT ABOUT
THE GRANT PROGRAM.
WE HAVE HAD A RARE DISEASE GRANT
PROGRAM FOR A NUMBER OF YEARS
WHERE WE FUND NATURAL HISTORY
STUDIES AS WELL AS SOMETIMES
CLINICAL TRIAL TRANSLATIONAL
RESEARCH.
THIS IS A NEW GRANT PROGRAM
FOCUSING ON A.L.S. AND OTHER
RARE NEURODEGENERATIVE DISEASES.
SO WE NEEDED TO DEFINE OUR
INVESTMENT PRIORITIES REACHING
OUT TO THE CENTERS FOR
REGULATORY SCIENCE, PRIORITIES
WE SAW IN DRUG DEVELOPMENT,
FIRST WITH OUR EE QIF LAN SEE
STUDIES FOR HE RAO MOTOR USE
CLINICAL OUT COME ASSESSMENTS.
AGAIN, THIS IS REALLY THINKING
ABOUT HOW DO WE MAKE OUR TRIALS
MORE ACCESSIBLE AND POTENTIALLY
DECENTRALIZED.
CERTAINLY NATURAL HISTORY
STUDIES, IMPROVING OUR
UNDERSTANDING OF THESE DISEASES
LOOKING FOR BIOMARKERS,
POTENTIALLY EVEN HAVING THESE
STUDIES SERVE AS EXTERNAL
CONTROLS IN APPROPRIATE
SITUATIONS.
LOOKING AT DEVICES FROM OUR
DEVICE -- WE ISSUED THREE GRANTS
AND TWO CONTRACTS THAT ALIGN
WITH THE GOALS OF THE PROGRAM.
WE WANT TO THANK NIH AND IN
PARTICULAR NIAMS FOR
CONTRIBUTING TO THE A.L.S.
NATURAL HISTORY GRANT AND THE
REMOTE USE EQUIVALENCY STUDY.
I THINK MOST PEOPLE ARE AWARE
THAT THE A.L.S. FRS IS THE
CLINICAL OUTCOME ASSESSMENT
MEASURE THAT'S USED IN OUR
CLINICAL TRIALS.
IN ADDITION, AGAIN, TAKING INTO
CONGRESS' ASK THAT WE ALSO LOOK
AT OTHER RARE NEURODEGENERATIVE
DISEASE, WE FUNDED A MYOTONIC
DYSTROPHY TYPE 1 NATURAL HISTORY
AND ATAXIA TELANGIECTASIA.
ALSO OUR COLLEAGUES IN THE
DEVICE CENTER ARE, AGAIN,
STARTING THEIR WORK IN THE
LANDSCAPE ANALYSIS OF BRAIN
COMPUTER INTERFACE FOCUSED
PATIENT PREFERENCE STUDIES IN
A.L.S.
AFTER FUNDING THESE, WE OPENED A
DOCKET TO ASK OUR STAKEHOLDERS
FOR FUNDING CONCEPTS FOR FY 23.
WE ALSO PUBLISHED AN RFI FOR A
POSSIBLE CONTRACT IN FY 23 TO
CONDUCT A BRAIN CENTERS COMPUTER
INTERFACE LANDSCAPE ANALYSIS FOR
CLINICAL OUT COME ASSESSMENTS.
FOR OUR PLANS FOR THIS YEAR,
THROUGH THE DOCKET.
SOMDOCKET, SOME OF THEINPUT WE D
PATHOPHYSIOLOGY AND ADVANCED
BIOMARKER DEVELOPMENT.
THERE WAS SOME COMMENTS ABOUT
SUPPORTING CLINICAL TRIALS BUT
THE FUNDING FOR THIS GRANT
PROGRAM, WE DON'T ANTICIPATE TO
BE ABOVE A COUPLE MILLION, WHICH
DOES MAKE IT DIFFICULT TO DO ANY
LARGE CLINICAL TRIALS.
SO WE WILL BE ISSUING ANOTHER
RFA ON NATURAL HISTORY AND
BIOMARKER STUDIES, AS WELL AS
FOLLOWING UP ON OUR WORK ON THE
BRAIN COMPUTER INTERFACES AND
CONTINUING SUPPORT FOR THE
MYOTONIC DYSTROPHY STUDY.
WE'VE ALSO BEEN ASKED BY
DR. WALK FOR SOME ADDITIONAL
FUNDING TO EXPAND THAT STUDY.
AGAIN WE'LL BE LOOKING AT THAT
PROPOSAL AS WELL AS OUR FUNDING.
THE OTHER THING WE WERE ASKED TO
DO UNDER THE ACT WAS TO PUBLISH
AN ACTION PLAN FOR RARE
NEURODEGENERATIVE DISEASES
INCLUDING A.L.S.
THIS HAPPENED SIX MONTHS INTO
THE PROGRAM.
WE POSTED THIS ON OUR WEBSITE.
IT'S A FIVE-YEAR ACTION PLAN.
IT TALKS ABOUT ADDRESSING --
WHAT WE'LL DO TO ADDRESS THE
UNMET MEDICAL NEEDS.
AS OUR FIRST PART OF THIS, WE
ISSUED AN A.L.S. SCIENCE
STRATEGY, AGAIN RECOGNIZING THAT
THIS ACT WAS DRIVEN IN LARGE
PART TO ADDRESS A.L.S. AND DRUG
DEVELOPMENT, SO WE DEVELOPED A
DISEASE-SPECIFIC APPROACH AND
ALSO REITERATED THAT, OF COURSE,
IT'S A MULTIPRONGED APPROACH,
FDA WORKING BOTH INTERNALLY AS
WELL AS EXTERNALLY WITH OUR
STAKEHOLDERS THROUGH RESEARCH
AND WITH OUR OTHER GOVERNMENT
AGENCIES.
THIS IS JUST SOME OF THE
ACTIVITIES WE PROMISED TO DO,
SOME OF WHICH WE'VE ALREADY
ENACTED, ONE WAS TO ESTABLISH AN
FDA RARE NEURODEGENERATIVE
DISEASES TASK FORCE.
I'LL TALK A LITTLE ABOUT HOW
WE'VE DONE THAT.
BUT ALSO TO LEVERAGE SOME OF THE
OTHER INITIATIVES THAT WE HAVE
INCLUDING OUR ACCELERATING RARE
DISEASE CURES PROGRAM THAT WE'VE
LAUNCHED WITHIN THE PAST YEAR.
TO ESTABLISH PUBLIC-PRIVATE
PARTNERSHIP FOR RARE
NEURODEGENERATIVE DISEASES WITH
NIH, I'LL TALK A LITTLE ABOUT
WHAT WE'VE DONE THERE TO
FACILITATE AND SUPPORT RESEARCH
AND INNOVATION, IN DRUG
DEVELOPMENT TOOLS AMONG OTHER
ACTIVITIES AND TO FOSTER
EXTERNAL ENGAGEMENT WITH A RARE
NEURODEGENERATIVE DISEASE.
AND FINALLY, DEVELOP
DISEASE-SPECIFIC SCIENCE
STRATEGIES, DECIDED TO FOCUS ON
THE A.L.S. SCIENCE STRATEGY AND
WE'LL DEVELOP MORE AS WE SEE THE
NEED AND RESOURCES ARE
AVAILABLE.
SO A LITTLE BIT ON THE SCIENCE
STRATEGY.
WE HAVE HAD A NUMBER OF
ACTIVITIES IN THE PAST YEAR TO
SUPPORT DRUG DEVELOPMENT FOR
AMYOTROPHIDEVELOPMDEVELOPMENT FE
BACK IN 2019 FOR DRUG
DEVELOPMENT, SOME WORKSHOPS WE
DID IN 2020 IN COOPERATION WITH
DUKE MARGOLIS TO HIGHLIGHT
PROGRESS AND LOOK FOR HOW WE
ADVANCE THESE PRODUCTS.
A 2021 GUIDANCE BY THE CENTER
FOR BIOLOGICS ON GENE THERAPY
FOR NEURODEGENERATIVE DISEASES,
AND OF COURSE WE CONTINUE TO
WORK WITH A.L.S. CAREGIVERS,
RESEARCHERS AND DRUG
DEVELOPMENT.
THE SCIENCE STRATEGY REALLY
BUILDS ON THE CURRENT SCIENCE
AND RESEARCH TO SUPPORT DRUG
DEVELOPMENT AND TO INFORM THAT,
WE REALLY LOOKED AT OUR
PORTFOLIOS AND LOOKED AT WHERE
WE HAD UNMET MEDICAL NEED, WHERE
THERE WERE CHALLENGES IN DRUG
DEVELOPMENT AND WHAT WE COULD
DO.
AND I THINK THERE'S REALLY THREE
AREAS OF FOCUS, I'LL DO A LITTLE
BIT OF A DEEPER DIVE, TO IMPROVE
CHARACTERIZATION OF THE
PATHOGENESIS AND NATURAL
HISTORY.
I THINK WE ALL AGREE THAT THAT'S
AN IMPORTANT STEP TO FACILITATE
ACCESS TO INVESTIGATIONAL NEW
DRUGS, AND TO ENHANCE CLINICAL
TRIALS, AGILITY, INFRASTRUCTURE.
SO THIS IS MORE OF A SCHEMATIC
OF HOW WE LAID OUT, OBVIOUSLY,
SOME OF THE EARLY AREAS AS TO
THE FDA RARE NEURODEGENERATIVE
TASK FORCE AND TO FOCUS SOME OF
THAT WORK ON A.L.S.
TO SUPPORT TRANSLATIONAL SCIENCE
RESEARCH, AND IN MANY CASES THAT
MEANS MAKING SURE WE'RE DOING
APPROPRIATE OUTREACH TO THOSE
THAT ARE DEVELOPING DRUGS,
ESPECIALLY THOSE PERHAPS IN
ACADEMICS WHO MAY NOT BE AS
FAMILIAR WITH THE REGULATORY
REQUIREMENTS, THAT THEY
UNDERSTAND THE PATHWAY TO
TRANSLATE THEIR SCIENCE INTO
DRUG DEVELOPMENT.
AGAIN, AS I SAID, A FOCUS ON
AMYOTROPHIC LATERAL SCLEROSIS
NATURAL HISTORY, COLLECTING
PATIENT PERSPECTIVES ON CLINICAL
TRIAL PARTICIPATION, HOWL WE CAN
MAKE THOHOW WE CANMAKE THOSE MOD
EASIER FOR PATIENTS TO
PARTICIPATE IN.
WE WANT TO ENCOURAGE DATA
SHARING AND IN PARTICULAR
DEVELOP STUDY DATA STANDARDS FOR
AMYOTROPHIC LATERAL SCLEROSIS.
THE CELL AND GENE THERAPIES
SAFETY PROJECT IS ONE WHERE
THEY'RE LOOKING ACROSS BOTH
A.L.S. AND OTHER
NEURODEGENERATIVE DISEASES FOR
GENE THERAPY TO LOOK FOR COMMON
SAFETY ISSUES.
EXPLORING THE USE OF
FIT-FOR-PURPOSE DIGITAL HEALTH
TECHNOLOGIES, AND ENCOURAGING
THE INCORPORATION OF EXPANDED
ACCESS INTO CLINICAL
DEVELOPMENT, ALSO PROMOTING
SUCCESSFUL TRANSITION -- AND THE
FUNDING FROM NIH WILL HELP THOSE
CONVERSATIONS, ESPECIALLY FOR
THE SMALL BUSINESSES AND JUST
PROMOTING SUCCESSFUL BENCH TO
BEDSIDE.
IF WE'RE SUCCESSFUL IN SOME OF
THESE ISSUES, WE HOPE TO START
TO CONTINUE TO INNOVATE CLINICAL
TRIAL DESIGNS AND ENHANCE
CLINICAL TRIAL INFRASTRUCTURE
AND AGILITY.
OUR CENTER FOR DEVICES, OF
COURSE WE HAVE A NUMBER OF
DEVICES THAT HELP PATIENTS LIVE
WITH A.L.S. BUT REALLY THEY WANT
TO FOCUS ON THE NEXT BRAIN
COMPUTER INTERFACES TO ENABLE
COMMUNICATION WHEN THAT IS NO
LONGER POSSIBLE.
AS WELL AS DIAGNOSTICS, WE THINK
OF MRIs, BUT HOW CAN WE MOVE
FORWARD WITH IN VITRO
DIAGNOSTICS TO PROVIDE EARLIER
DIAGNOSIS, ENABLING EARLIER
TREATMENT TO DETECT BIOMARKERS
THAT MAKE A PATIENT LIKELY TO
RESPOND TO A SPECIFIC TREATMENT,
WHICH REALLY STREAMLINES DRUG
DEVELOPMENT AND TO MONITOR
DISEASE PROGRESSION TO HELP
GUIDE TREATMENT.
SO DIGITAL HEALTH S IS ANOTHER
PRIORITY AND I THINK IT'S
SELF-EVIDENT THAT REMOTE DATA
COLLECTION CAN REALLY HELP US TO
DECENTRALIZE CLINICAL
INVESTIGATIONS AND IMPROVE
ACCESS TO CLINICAL
INVESTIGATIONS BY BRINGING MORE
OF THE CLINICAL TRIAL TO WHERE
THE PATIENT IS AND POTENTIALLY
FEWER VISITS TO STUDY SITES.
IT MAY ALSO FACILITATE
INNOVATIVE CLINICAL
INVESTIGATION END POINTS GOING
BEYOND THE ONES WE HAVE TODAY,
AND THEN FACILITATING CAPTURING
REAL WORLD DATA AND PATIENT
GENERATED HEALTH DATA SO WE CAN
REALLY UNDERSTAND A
PARTICIPANT'S DAILY LIFE AND
LONGITUDINAL FOLLOW-UP AND A
MORE DETAILED PICTURE OF THE
IMPACT OF THE MEDICAL CONDITION
ON A PATIENT.
AS I SAID, WE FORMED A TASK
FORCE FOR NEURODEGENERATIVE
DISEASE FOR REPRESENTATION FROM
OUR CENTERS FOR DRUGS,
BIOLOGICS, DEVICES AND THE
OFFICE OF THE COMMISSIONER.
WE WANT TO COORDINATE INPUT FOR
THE PUBLIC-PRIVATE PARTNERSHIP
AND OTHER ACTIVITIES RELATED TO
THE IMPLEMENTATION OF THE ACT
FOR A.L.S., INCLUDING REGULATORY
SCIENCE PRIORITIES AND TO ENSURE
THAT WE HAVE A COHESIVE FDA
APPROACH TO AND COORDINATED
EFFORTS TOWARDS ADVANCING
SCIENCE AND DRUG DEVELOPMENT FOR
RARE NEURODEGENERATIVE DISEASES
SO WE'RE COORDINATED ON
CROSS-CUTTING CLINICAL AND
REGULATORY POLICY ISSUES TO
FOSTER CONSISTENCY, SOMETHING
THAT'S REALLY ALWAYS ASKED OF US
AS WE MOVE FORWARD.
WE ALSO WANT TO MAKE SURE WE
LEVERAGE SOME OF OUR OTHER
PROGRAMS TO MAKE THINGS
SYNERGISTIC RATHER THAN SILOED.
SOME OF YOU MAY OR MAY NOT BE
AWARE IN 2022, WE LAUNCHED THE
ACCELERATING RARE DISEASE CURES
PROGRAM IN CDER TO DRIVE
SCIENTIFIC AND REGULATORY
INNOVATION AND HE GAUGEMENT.
MANY OF YOU MAY KNOW THAT WE
HAVE HAD AN INCREASE IN DRUG
DEVELOPMENT FOR RARE DISEASES,
IN SOME YEARS CLOSE TO 50% OF
OUR NEW MOLECULAR ENTITIES ARE
FOR A RARE DISEASE BUT THERE'S
MUCH MORE TO BE DONE.
AGAIN, THIS IS THE CENTER FOR
DRUG PROGRAM, OFFICE OF THE
CENTER DIRECTOR, OFFICE OF
TRANSLATIONAL SCIENCE, OFFICE OF
NEW DRUGS.
OUR VISION IS THAT IT REALLY
WILL BE ACROSS FDA WITH INPUT
FROM OUR OTHER CENTERS AND OTHER
OFFICES.
SOME OF THE THINGS IT'S REALLY
FOCUSED TWO WAY, ONE, OUTREACH
AND EDUCATION, ONE OF THE THINGS
THAT WE WILL BE WORKING ON IS
LEARNING IN AND EDUCATION TO
ADVANCE AND EMPOWER RARE
DECEMBER DRUG DEVELOPERS, MAKING
SURE THAT THOSE WHO ARE
TRANSLATING THEIR RESEARCH INTO
CLINICAL TRIALS REALLY
UNDERSTAND HOW TO MAKE THAT
TRANSITION INTO GETTING A
PRODUCT LICENSED.
SO YOU KNOW, WE'RE SEEKING INPUT
FROM KEY STAKEHOLDERS IN TERMS
OF WHAT THEY UNDERSTAND AND
DON'T UNDERSTAND ABOUT THE DRUG
DEVELOPMENT PROCESS FROM AN FDA
PERSPECTIVE.
WE'VE BEGUN OUTREACH TO OUR U.S.
GOVERNMENT STAKEHOLDERS AND
WE'VE OPENED A DOCKET FOR PUBLIC
COMMENTS TO INFORM THIS
ACTIVITY.
OF COURSE YOU KNOW, ACTIVITIES
SUCH AS A RARE DISEASE, WE'LL BE
PRESENTING ON SOME OF THE
CHALLENGES OF CLINICAL TRIALS
WITH SMALL POPULATIONS,
IMPORTANT CONSIDERATIONS, AS
WELL AS PRESENTING ON THE ARC
PROGRAM, AND THE PROGRAM WAS
FEATURED IN A DECEMBER ARTICLE
IN END POINT NEWS, IF ANYONE
WANTS MORE INFORMATION.
THERE'S ALSO A LOT OF INTERNAL
ACTIVITIES, AGAIN, CREATING AN
INTERNAL KNOWLEDGE RESOURCES TO
UNDERSTAND INNOVATIVE
APPROACHES, AND MAKE SURE WE'RE
LEVERAGING EXPERTISE ACROSS
DIVISIONS JUST LIKE NIH, WE HAVE
A LOT OF DIFFERENT DIVISIONS AND
OFFICES.
WE WANT THOSE WITH THE MOST
EXPERTISE TO BE ABLE TO CONSULT
ON EACH APPLICATION AND ENSURE
CONSISTENCY AND THAT WE'RE BEING
AS INNOVATIVE AS WE SHOULD BE.
WE WANT TO CONTINUE INTERNAL
FORUMS THAT REALLY DISCUSS THE
CHALLENGING APPLICATIONS AND
POLICY, AND THEN HAVE A COMMON
INPUT INTO OUR REGULATORY
SCIENCE PRIORITIES AND MAKE SURE
WE'RE GETTING RESULTS FROM THOSE
INVESTMENTS.
IN TERMS OF THE PRIVATE-PUBLIC
PARTNERSHIP -- PUBLIC-PRIVATE
PARTNERSHIP, AS MANY OF YOU MAY
KNOW, IN SEPTEMBER, NIH AND FDA
LAUNCHED THIS PUBLIC-PRIVATE
PARTNERSHIP FOR RARE
NEURODEGENERATIVE DISEASES.
WE SELECTED THE CRITICAL PATH
INSTITUTE AS THE CONVENER OF THE
PARTNERSHIP.
FOR MANY OF YOU, CRITICAL PATH
HAS WORKED WITH FDA EXTENSIVELY
ON REGULATORY SCIENCES AND NIH
IS ALSO PARTICIPATED IN THEIR
ACTIVITIES.
THEY HAVE CONCENTRATION AREAS
FOR THIS PART OF THE
PUBLIC-PRIVATE PARTNERSHIP WILL
BE THE NEUROLOGY PROGRAM, THEIR
PEDIATRIC PROGRAM, AS WELL AS
THEIR RARE ORPHAN DISEASE
PROGRAM.
THEY ALREADY HAVE A CONSORTIA,
HUNTINGTON'S DISEASE, RS --
REGULATORY SCIENCES CONSORTIUM.
THE CPTA IS THE CRITICAL PATH
THERAPEUTICS FOR ATAXIA.
THEIR CORE COMPETENCIES ARE IN
REGULATORY SCIENCE, THE COA
PROGRAM, THEY WORK ON QUALIFYING
DIFFERENT PROs AND CLINICAL
OUTCOME ASSESSMENTS FOR
REGULATORY USE, AS WELL AS
BIOMARKERS, THEY'VE BEEN
INSTRUMENTAL IN DEVELOPING SOME
BIOMARKERS THAT HAVE QUALIFIED
FOR UNDER FDA'S BIOMARKER
QUALIFICATION PROGRAM.
THEIR QUANTMED PROGRAM IS
FOCUSED ON MODEL INFORMED DRUG
DEVELOPMENT AND THEIR DATA
COLLABORATION, THE DCC, IS
REALLY FEEDING INTO THE RARE
DISEASE CURES ACCELERATOR
ANALYSIS PROGRAM, WHICH IS WHAT
WE CALL RDCA DAP, SOMETHING THAT
WAS FORMED WITHIN NIH TO REALLY
BRING DATA TOGETHER.
I HAVE A SLIDE ON THAT AND I'LL
TALK HOW WE REALLY WANT TO
INTEGRATE A.L.S. DATA INTO THAT
INITIATIVE.
SO THIS IS REALLY THE LEARN AND
CONFIRM, TAKING THE STRENGTHS OF
BOTH PARTNERS, NIH TRANSLATING
THE CLINICAL AND PRE-CLINICAL
DISCOVERY INTO RND AND THEN
ACCELERATING INNOVATION AND
REGULATORY SCIENCE FOR DRUG
DEVELOPMENT.
THIS IS JUST THE SCHEMATIC OF
THE RDCA-DAP, DATA COLLABORATION
CENTER, AND ABLE TO INGEST
MULTIPLE SOURCES OF DATA, CURATE
THEM INTO A STANDARDIZED DATA
STANDARD AND REFORMAT AND CLEAN
SO YOU HAVE FULLY CURATED AS
WELL AS MINIMAL LIQUEUR RATED
THAT CAN BE FED INTO OTHER
INITIATIVES INCLUDING THE
QUANTITATIVE MEDICINE PROGRAM AS
WELL AS TO BE USED BY OTHER
RESEARCHERS.
I THINK THE LAST TIME I CHECKED
IN TERMS OF HOW MUCH DATA THEY
ACTUALLY HOLD, THEY HAVE ABOUT
230 DATASETS IN CLINICAL TRIALS,
SO I THINK THERE'S A LITTLE
MORE, OBSERVATIONAL STUDIES IN
REAL WORLD STUDIES.
A NUMBER OF DIFFERENT
NEUROLOGICAL DISEASES.
AS WELL AS -- AND THAT'S
ABOUT -- OVER ABOUT 175,000
PATIENT INFORMATION.
SO WE HOPE TO REALLY MOVE
FURTHER INTO OTHER RARE
NEURODEGENERATIVE DISEASES.
THEY DO HAVE PARKINSON'S AND
HUNTINGTONS RIGHT NOW, BUT WE'LL
EXPAND THAT INTO AMYOTROPHIC
LAA.L.S.SOME OF THE THINGS THATE
GOING ON, WE ARE WORKING ON A
STAKEHOLDER MAP UNDER
DEVELOPMENT, AND ALSO WORKING ON
A MEETING THAT WILL BE
SPECIFICALLY, YOU KNOW, OUT TO
OUR STAKEHOLDERS TO UNDERSTAND
WHERE THE GAPS ARE FOR A.L.S.
AND WHAT WE CAN DO IN THE
REGULATORY SCIENCE SPACE AS WELL
AS IN INDUSTRY.
IN THE HUNTINGTON'S, THERE'S
BEEN SUBMIGS FOR QUANTITATIVE
CLINICAL TRIAL SIMULATION, IN
THE ATAXIA, THERE IS CONTINUITY
FOR COA-FOCUSED EFFORT, DATA
INTERFACING AND DISEASE STAGING
DEFINITION BEING WORKED ON, AS
WELL AS STRATEGY FOR FRIEDRICH'S
ATAXIA, AND FINALLY THINKING
ABOUT QUANTITATIVE, SOME A.L.S.
MODELING INVENTORIES UNDER
DEVELOPING AND PREPPING A DATA
STRATEGY FOR A.L.S. TO FEED INTO
THE RDCA-DAP.
SO I THINK IN SUMMARY, YOU KNOW,
THE ACT FOR A.L.S. LAYS OUT A
MULTIFACETED APPROACH TO
ADVANCING DRUG DEVELOPMENT FOR
A.L.S. AND OTHER RARE
NEUROLOGICAL DISEASES.
WE'RE ENGAGING ON MULTIPLE
FRONTS BOTH EXTERNALLY AND
INTERNALLY, AND WE REALLY LOOK
FORWARD TO, WOULDING WITH NIH
AND OUR PUBLIC-PRIVATE
PARTNERSHIP TO ADVANCE NEW
THERAPIES THAT WILL ADDRESS
SIGNIFICANT UNMET NEEDS.
AND I WANT TO THANK YOU FOR YOUR
ATTENTION.
>>THANK YOU FOR THIS REALLY
EXCELLENT OVERVIEW.
UNLESS WE HAVE QUESTIONS FROM
COUNCIL, I'LL THEN TURN IT OVER
TO WALTER.
>>WELL, THANK YOU VERY MUCH,
JACQUELINE.
IT'S REALLY EXCITING TO BE
WORKING WITH THE FDA AND I THINK
IT WAS JUST SO COMPLEMENTARY,
THE FDA WORKING WITH INDUSTRY IN
TERMS OF GETTING PRODUCTS
APPROVED, KNOWING THAT THEY
WORK, GETTING THEM APPROVED.
NIH GENERALLY IS WAY KIND OF
BACK WHERE WE'RE DOING THE
SCIENCE THAT ENABLES THE
INDUSTRY TO IDENTIFY TARGETS,
MAYBE VALIDATE THEIR TARGETS,
THINK ABOUT HOW TO DO PHASE
2 TRIALS, BUT USUALLY AFTER
THAT, IT'S KIND OF HANDS OFF AND
WE HAVE LITTLE INTERACTION WITH
THEM AS THEY MOVE TOWARDS THE
REALLY CENTRAL PIECE, WHICH IS
GETTING THINGS APPROVED IN A
REGULATORY SCIENCE, AND SO I
THINK WE'RE GOING TO LEARN A
LOT, I THINK, FROM WORKING WITH
YOU.
WE REALLY LOOK FORWARD TO IT.
I WANTED TO JUST TALK A LITTLE
BIT ABOUT THE STRATEGIC PLAN
AGAIN, SO IF I COULD HAVE MY
FIRST SLIDE.
THIS IS JUST A REFERENCE BACK TO
WHAT WAS PRESENTED BY THE
WORKING GROUP.
THESE ARE THE KIND OF MAJOR
AREAS, UNDERSTANDING THE BIOLOGY
BEHIND AMYOTROPHIC LATERAL A.L.F
A STRONGSUIT OF NINDS.
TRANSLATING THE FUNDAMENTAL
RESEARCH INTO POTENTIAL A.L.S.
THERAPIES.
FOR US, THAT'S USUALLY GETTING
THINGS TOWARDS THE PHASE TWO
STAGE.
BUT USUALLY NOT INVOLVED IN THAT
FINAL STAGE OF GETTING THINGS TO
THE FDA FOR APPROVAL.
I WOULD SAY ONE AREA WE'VE BEEN
VERY ACTIVE IN, WHICH JACQUELINE
MENTIONED, WAS THE
BRAIN-COMPUTER INTERFACE STORY.
PEOPLE MAY HAVE SEEN SOME OF THE
LITERATURE IN THE BRAIN
INITIATIVE PROJECTS, PEOPLE WITH
A.L.S. WHO ARE UNABLE TO SPEAK
ARE ABLE TO COMMUNICATE WITH
THESE REALLY SOPHISTICATED
BRAIN-COMPUTER INTERFACES, AND
SO ONE BIG CHALLENGE NOW, I
THINK, IS ACTUALLY GETTING THEM
TO THE FDA FOR THEIR
DISSEMINATION AND USE.
IN THE
COMMERCIAL SPACE.
WE WORK VERY MUCH IN TANDEM WITH
FDA IN TERMS OF OPTIMIZING THE
A.L.S. CLINICAL RESEARCH.
WE MAY BE MORE PHASE
TWO-CENTERED THAN PHASE THREE
CENTERED, FDA BEING MORE PHASE
THREE CENTERED.
WE ALL WANT TO IMPROVE THE
QUALITY OF LIFE, AND I THINK
THAT THE STRATEGIC PLAN REALLY,
IF ONE THING IT TAUGHT US IS
THAT THERE'S SO MANY PEOPLE
WORKING ON THE SAME GOAL THAT
IDENTIFY A WAY OF BRINGING
PEOPLE TOGETHER FOR
COLLABORATIONS AND PARTNERSHIPS,
AND PUT SOME KIND OF A
CENTRALIZED PROCESS TOGETHER
WHERE PEOPLE CAN SEE WHAT'S
GOING ON, KNOW WHERE THE
PROGRESS IS, WHERE THE GAPS ARE,
I THINK THAT WILL HELP EVERYONE
GOING FORWARD.
SO GOING FORWARD, I THINK THE
STRATEGIC PLAN GIVES US A LOT OF
REALLY GOOD INFORMATION ON HOWL
HOWWE CAN PLAN HERE AT NINDS.
NOW DAN AND JACQUELINE TALKED
ABOUT THE BILL, AND SO THE BILL
HAS AS ITS -- THE BILL HAS AS
ITS GOAL TO DIRECT THE SECRETARY
OF HEALTH AND HUMAN SERVICES TO
SUPPORT RESEARCH ON EXPANDED
ACCESS TO INVESTIGATIONAL DRUGS
FOR A.L.S. AND FOR OTHER
PURPOSES.
SO WE HAVE A PROGRAM NOW WHERE
WE ARE ACTUALLY OPEN NOW.
WE STARTED JANUARY 9 OPEN FOR
APPLICATIONS TO COME IN FOR
GRANTS TO UTILIZE EXPANDED
ACCESS, AND WE WOULD FUND THE
MANUFACTURE AND THE
DISSEMINATION OF DRUGS, AND THE
RESEARCH THAT WOULD GO ON TO
UNDERSTAND A LOT OF THE
QUESTIONS THAT WERE POSED BY THE
WORKING GROUP AND THE STRATEGIC
PLAN ABOUT THINGS LIKE
BIOMARKERS, NATURAL HISTORY,
THROUGHOUT THE STAGES OF THE
DISEASE.
BUTTON NUMBER TWO IS THE
PUBLIC-PRIVATE PARTNERSHIP FOR
RARE NARROW DEGENERATIVE
DISEASES BETWEEN THE NIH AND
FDA.
YOU HEARD A LOT OF ABOUT THAT
FROM JACQUELINE.
SO THIS CALLS FOR PARTNERSHIPS
AND CONSORTIA WITH OTHER PUBLIC
AND PRIVATE ENTITIES WITH
EXPERTISE IN AMYOTROPHIC LATERA.
SO ONE THING WE'RE HOPING IS TO
BRING EVERYONE TOGETHER, LIKE I
JUST MENTIONED EARLIER, SO
EVERYBODY KIND OF KNOWS WHAT'S
GOING ON, WHAT THE GAPS ARE AND
WHERE WE CAN ACTUALLY PARTNER
AND COLLABORATE AND HAVE KIND OF
A CENTRAL PLACE WHERE ALL THIS
INFORMATION CAN COME.
FDA WILL BE FOCUSING ON
ADVANCING REGULATORY SCIENCE AND
THE RESEARCH THAT WILL SUPPORT
AND ACCELERATE THE REVIEW OF THE
DRUGS AND THE DEVELOPMENT.
I THINK NIH WOULD FOCUS MORE ON
THINGS THAT WOULD ACCELERATE THE
DEVELOPMENT OF NEW TARGETS,
VALIDATE NEW TARGETS, AND MAYBE
GET THEM INTO, YOU KNOW, KNOWING
WHAT POPULATION OF PATIENTS
WOULD BE BEST TO TEST IN A PHASE
2 TRIAL, THOSE KIND OF THINGS.
I THINK BOTH OF US WILL BE
INTERESTED IN LEARNING MORE
ABOUT THE NATURAL HISTORY, AND
ESPECIALLY GIVEN THE STRATEGIC
PLAN OF REALLY CONCENTRATING ON
FOLKS WHO ARE AT RISK FOR
DEVELOPING A.L.S.
CERTAINLY I WAS IN HUNDRED TINGS
DISEASE BEFORE COMING, AND THAT
WAS A REALLY BY PART OF HUNDRED
HUNTINGTON'S DISEASE, WE
ACTUALLY RAN EXPERIMENTS TO
ACTUALLY BE ABLE TO BE IN A
POSITION WHERE WE COULD TEST
THINGS IN PEOPLE WHO DO NOT HAVE
ANY MANIFESTATION OF THE
DISEASE.
YET THE IDEA WAS THAT WE WOULD
LEARN HOW TO SET UP CLINICAL
TRIALS THAT WOULD PREVENT THE
ONSET OF THE DISEASE.
SO I THINK THAT'S WHAT'S COMING
TO A.L.S.
THAT'S SOMETHING WE WOULD REALLY
LIKE TO DO.
WE WANT TO IDENTIFY THE
MOLECULAR TARGETS.
AND THEN THE OTHER CALL WAS TO
INCREASE THE EFFICIENCY OF
CLINICAL DEVELOPMENT OF
THERAPIES IN THE ESTABLISHMENT
OF CLINICAL TRIAL NETWORKS.
THE PARTNERSHIP ALLOWS US TO
WORK WITH GROUPS SUCH AS THE
CLINICAL PATH INSTITUTE, AND
WE'RE GOING TO BE TALKING ABOUT
THE FOUNDATION OF NIH, WHERE
THEY HAVE THE POWER UNDER THIS
BILL TO SOLICIT AND ACCEPT GIFTS
AND GRANTS, AND OTHER DONATIONS
TO KIND OF COORDINATE THIS KIND
OF ACTIVITY.
AND THEN THE FDA, JACQUELINE
TALKED TO YOU ABOUT THE RARE
NEURODEGENERATIVE DISEASE GRANT
PROGRAM.
SO JACQUELINE ALSO TALKED ABOUT
THE PUBLIC-PRIVATE PARTNERSHIP
THAT'S ESTABLISHED ALREADY, AND
THAT'S WITH THE NIH AND THE FDA,
AND PARTICULARLY WITH THE
CRITICAL PATH INSTITUTE AND
NINDS HAS HAD REALLY GOOD
RELATIONSHIPS WITH THE CRITICAL
PATH INSTITUTES.
I KNOW JANE WAS ACTUALLY AT THE
CLINICAL PATH INSTITUTE PRIOR TO
COMING ON COUNCIL.
AND I THINK THERE'S A LOT THAT
WE CAN DO TOGETHER IN THIS
SPACE, PARTICULARLY ON
ACCELERATING THE INNOVATION IN
REGULATORY SCIENCE BEHIND THE
R & D.
WHAT WE'D LIKE TO DO ALSO IS TO
WORK WITH OUR PARTNERS AT THE
FOUNDATION FOR NIH TO DEVELOP A
PORTAL OF HARMONIZING DATA
COMING IN FROM ALL SEGMENTS OF
A.L.S. RESEARCH.
THIS, OF COURSE, WOULD BE
VOLUNTARY FOR THE NON-PROFIT
ORGANIZATIONS, BUT HOPING THEY
CAN SEE THE BENEFIT OF BRINGING
THEIR DATA IN TO A SINGLE
PORTAL.
WE AT NIH WOULD PROBABLY VERY
SOON MANDATE THAT ALL OUR
GRANTS, THE DATA WOULD HAVE TO
COME IN TO SUCH A PORTAL, AND I
THINK THE FDA COULD CONSIDER
SIMILARLY DOING THAT.
AND THAT IS -- IT'S SOMEWHAT
BASED ON THE EXPERIENCE WE'VE
HAD WORKING WITH THE FNIH IN
PARKINSON'S DISEASE, WHERE WE
HAVE THIS REALLY TREMENDOUS DATA
PORTAL FOR ALL THE CLINICAL
DATA.
THIS IS CLINICAL DATA,
PATIENT-LEVEL DATA AND
PARKINSON'S ALL COMING TOGETHER.
AND THE CONSORTIUM OF PEOPLE WHO
WOULD POTENTIALLY BE INVOLVED
AND HOPEFULLY SEE VALUE WOULD BE
THE ACADEMIC A.L.S.
INVESTIGATORS, THE FDA, THE
CRITICAL PATH INSTITUTE,
PHARMACEUTICAL COMPANIES,
NON-PROFIT ORGANIZATIONS,
BIOTECH COMPANIES, THE
FOUNDATION FOR NIH, AND NIH
ITSELF.
AND HIT THE BUTTON ONCE MORE.
AND WHAT WE'VE DONE IN
PARKINSON'S AND WHAT WE WOULD
THINK ABOUT DOING IN A.L.S. IS
DEVELOPING A GROUP THAT WOULD GO
OUT AND COLLECT DATA AND COLLECT
SAMPLES AND DEVELOPING A
BIOREPOSITORY WHERE BLOOD
SAMPLES, SPINAL FLUID SAMPLES,
POTENTIALLY TISSUE SAMPLES COULD
BE STORED TO DO REALLY IN DEPTH
CLINICAL PHENOTYPING OF THE
DIFFERENT TYPES OF A.L.S.,
TRYING TO UNDERSTAND THE DISEASE
PROGRESSION, WHAT DETERMINE AS
SLOW PROGRESSION FROM A FAST
PROGRESSION, GETTING OLD GENOME
SEQUENCING ON HUNDREDS AND
THOUSANDS OF PEOPLE COLLECTING
CSF AND BLOOD, NOT ONLY
COLLECTING IT BUT ANALYZING IT
FOR, SAY, TRANSCRIPTOMICS OR
PROTEOMICS.
ONE BIG AREA NOW THAT'S
EXPLODING IN NEURODEGENERATIVE
DISEASE SCIENCE IS EXAMINING
SPINAL CORD AND BRAIN AT A
SINGLE CELL LEVEL.
THIS CAN BE DONE IN AUTOPSY
TISSUE NOW, AND YOU CAN
UNDERSTAND WHAT IS ACTUALLY
GOING ON AT THE CELL LEVEL, AS
THE CELLS BECOME SICK.
AND THIS LEADS TO POTENTIAL NEW
THERAPEUTIC TARGETS THAT NO ONE
COULD EVER GET TO WITHOUT THIS
NEW TECHNOLOGY, WHICH ACTUALLY
CAME OUT OF THE BRAIN
INITIATIVE.
OTHER THINGS IMPORTANT FOR
A.L.S. WOULD BE NEUROAND MUSCLE
IMAGING.
GO BACK ONE SECOND.
IMAGING OF THE MUSCLE WHICH
ATROPHIES AND THE MOTOR CORTEX
AND MOTOR SYSTEM,
NEUROPHYSIOLOGY TESTING, AND
THEN THE INDUCED PLURIPOTENT
CELLS WHICH HAVE BEEN SO
VALUABLE IN STUDYING FORMS OF
A.L.S. IDENTIFYING REAL TARGETS
FOR THERAPIES AND THE BIG
QUESTION AS POSED BY THE
STRATEGIC WORKING GROUP, HOW TO
MOVE FROM THESE ADVANCES IN
GENETIC THERAPIES TO THE
SPORADIC A.L.S.
BUT TO HAVE -- THERE HAVE BEEN
SPLICING ABNORMALITIES THAT HAVE
BEEN FOUND RELATED IN THE
GENETIC FORM AND RELATED TO A
PROTEIN WHICH IS FOUND IN ALL
A.L.S., EVEN THE SPORADIC.
SO THERE HAVE BEEN THESE TIES
BETWEEN THE GENETIC FORMS AND
THE SPORADIC FORMS, AND THESE
CAN BE REALLY EXPLORED WITH
THESE INDUCED PLURIPOTENT CELLS
THAT CAN THEN BE TURNED INTO
MOTOR NEURONS.
COLLECTING THESE AND MAKING THEM
AVAILABLE FOR PEOPLE TO STUDY
WOULD BE SOMETHING THAT WE COULD
CONSIDER AS PART OF THIS BIG
PROGRAM.
SO IT ALL STARTS IF WE THINK
ABOUT THE IDEAS OF HOW THE DATA
WOULD FLOW, IT ALL STARTS WITH
PERSONS WITH A.L.S. OR PEOPLE
WHO ARE AT RISK FOR A.L.S. SO
THIS IS NOT BASIC NEUROSCIENCE,
THIS IS ALL CLINICAL WORK, AND
IT WOULD INCLUDE THE NIH AND THE
FDA FUNDED RESEARCH LIKE I
MENTIONED.
WE'RE HOPING WE COULD BRING IN
INDUSTRY TO BRING IN THEIR DATA,
CERTAINLY THE FDA AND THE
CRITICAL PATH INSTITUTE HAVE
BEEN VERY SUCCESSFUL THERE.
BUT ALSO TO ENGAGE NON-PROFITS
LIKE TARGET A.L.S. OR THE OTHER
A.L.S. ORGANIZATIONS THAT HAVE
DATA OR COULD HELP US IN THIS
GREAT EFFORT.
AND TO DO THIS, THOUGH, WE NEED
PEOPLE ON THE GROUND FLOOR,
WE'RE THINKING SETTING UP A
CONSORTIA OF SITES THAT WILL BE
ABLE TO COLLECT THIS KIND OF
DATA, WORK TOGETHER TOWARDS A
COMMON GOAL, AND I WOULD SAY
ALSO THAT ONE OF THE ISSUES WITH
EXPANDED ACCESS IS THAT THERE
ISN'T THE JIE GIANT WORKFORCE TE
ABLE TO MAKE THESE
INVESTIGATIONAL DRUGS AVAILABLE
ACROSS THE COUNTRY SO EXPANDING
THE WORKFORCE IN SUCH A
CONSORTIA WOULD ALSO HELP
GREATLY IN EXPANDING THE ACCESS
OF PATIENTS TO INVESTIGATIONAL
DRUGS WHICH WAS AND REMAINS THE
PRIMARY AIM OF THE A.L.S. BILL.
THE KEY TO THIS IS THAT THESE
DATA THAT EVERYONE IS COLLECTING
BECOMES COMPARABLE.
SO YOU NEED TO HARMONIZE THE
DATA.
WE'VE DONE THIS MULTIPLE TIMES
IN DIFFERENT DISEASES.
THIS IS A VERY HARD, LABORIOUS
AND EXPENSIVE PROCESS.
AND THERE ARE WAYS IN WHICH WE
CAN STREAMLINE IT GOING INTO THE
FUTURE.
EVEN THEN, IT REQUIRES A LOT OF
EFFORT TO MAKE SURE YOU HAVE
REALLY GOOD DATA WHEN YOU BRING
INTO YOUR PORTAL, AND THE IDEA
IS THAT WE CAN DO THAT AND THEN
BRING IT INTO THE PORTAL, MIGHT
BE A PORTAL THAT IS
FIT-FOR-PURPOSE FOR WHAT THE
FDA'S PURPOSES ARE AND ANOTHER
MIGHT BE FIT-FOR-PURPOSE WHAT
THE NIH'S PURPOSES ARE, MAYBE
MORE OF THE PHASE TWO EARLY
TARGET IDENTIFICATION COMPARED
TO THE REGULATORY SCIENCE.
SO THEY'RE VERY COMPLEMENTARY,
AND THEY HAVE TO HAVE COMMON
DATA SO YOU CAN GO BACK BETWEEN
ONE AND THE OTHER, BUT THIS
WOULD OFFER THE OPPORTUNITY TO
REALLY PUT THE EFFORTS OF BOTH
OUR AGENCIES TOGETHER TO SOLVE
THIS PROBLEM.
AND I THINK I'M GOING TO END UP
ON THIS SLIDE, AND BECAUSE I
THINK THAT A LOT OF THE A.L.S.
STRATEGIC PRIORITIES COULD BE
FURTHERED BY THIS TYPE OF
PUBLIC-PRIVATE PARTNERSHIP WHERE
WE'RE COLLECTING DATA, BRINGING
IT TOGETHER, COLLABORATION,
DECIDING ON WHAT DATA WE GET
NEXT, WHAT THINGS SHOULD WE
ANALYZE, BUT BRING ALL THE
RESULTS TOGETHER SO THAT THOSE
RESULTS ARE AVAILABLE TO
SCIENTISTS AROUND THE WORLD.
SO I THINK THAT THE CONSORTIA
THAT COLLECTS THE DATA WORKING
IN A VERY COLLABORATIVE BUT, YOU
KNOW, USING STANDARD OPERATING
PROCEDURES, WORKING UNDER MASTER
PROTOCOLS, ALL THE THINGS
MENTIONED IN THE STRATEGIC PLAN
WOULD REALLY ACCELERATE THE
ACQUISITION OF REALLY GOOD DATA,
AND THEN HARMONIZING IT WOULD
MAKE IT MUCH MORE VALUABLE THAN
IT IS NOW, AND I THINK WE CAN,
YOU KNOW -- I THINK THAT THE
PROJECT IS SO ATTRACTIVE THAT I
THINK MY GUESS IS THAT ANYONE IN
THE A.L.S. FIELD IS GOING TO
WANT TO BE PART OF THIS, AND WE
WANT THEM TO BE PART OF IT AND
WE'LL DO EVERYTHING WE CAN TO
MAKE THAT HAPPEN.
SO I'M REALLY EXCITED GOING
FORWARD WITH OUR NON-PROFIT
GROUPS, WITH THE FDA, NIH, AND
HOPING ALSO TO BRING INDUSTRY
INTO THIS MIX, BECAUSE THE OTHER
ONES WHO ARE GOING TO TELL US
WHAT THEY NEED TO GET THEIR
DRUGS OUT OF THEIR LABS AND
ACTUALLY INTO PEOPLE, AND THEN
WORKING WITH THE FDA, MOVING
FROM PHASE TWO TO PHASE THREE
AND HOPEFULLY TO APPROVALS.
SO I REALLY AM VERY OPTIMISTIC
AND KIND OF SEE FROM BEGINNING
TO END HOW THIS PUBLIC PRIVATE
PARTNERSHIP CAN GET US TO OUR
EVENTUAL GOAL, WHICH IS
EFFECTIVE THERAPIES FOR PEOPLE
WITH A.L.S. AND ACTUALLY
EFFECTIVE THERAPIES THAT CAN
PREVENT A.L.S. FROM SETTING IN
IN PEOPLE WHO ARE AT RISK.
SO THANKS FOR YOUR ATTENTION.
WE'RE REALLY INTERESTED IN ANY
COMMENTS.
WE'RE STILL WORKING THIS OUT,
AND SO ANY SUGGESTIONS ARE VERY
WELCOME AND YOU CAN ALWAYS EMAIL
ME.
GO TO THE LAST SLIDE.
THE LAST SLIDE JUST HAS MY EMAIL
AT THE BOTTOM, AND IT ALSO HAS
THE PEOPLE HERE IN OUR NINDS
A.L.S. WORKING GROUP, WHICH HAVE
BEEN, YOU KNOW, TREMENDOUS IN
WORKING ON NOT ONLY THE
STRATEGIC PLAN BUT ALSO ON THE
WORK WITH THE FDA AND THE
NASCENT PUBLIC-PRIVATE
PARTNERSHIP.
SO THANKS VERY MUCH FOR YOUR
ATTENTION.
>>OKAY.
WE DO HAVE -- WE'RE BEHIND TIME,
BUT I DON'T REALLY CARE.
WE DO HAVE TIME FOR A COUPLE OF
QUESTIONS.
I WISH WE HAD A LOT OF TIME TO
HAVE DISCUSSION BECAUSE THIS HAS
BEEN A WONDERFUL SEGMENT.
HANK?
HANK PAULSON.
>>AGAIN, VERY INSPIRING, ALL
THE PEOPLE THAT PRESENTED ON THE
STRATEGIC PLAN, I REALLY
APPRECIATE THAT.
AND THANK YOU, WALTER.
THINGS I HEARD WERE CENTERS OF
EXCELLENCE, THE NEED FOR
BIOMARKERS, THE NEED FOR A
NETWORK OF CLINICAL TRIAL SITES.
THERE ARE SO MANY DIFFERENT
MODELS OF RESEARCH CENTERS OF
EXCELLENCE, RESEARCH CENTERS,
ALZHEIMER'S FTD, HD, ATAXIA.
YOU CAN'T DO EVERYTHING IF YOU
WANT TO MEET THIS MOLECULAR
CLOCK, THERE'S AN URGENT TIME
PRESPRESSURE TO GET TRIALS UNDE.
SO I WOULD ENVISION THAT WHAT
YOU WOULD SEE AMONG THE CENTERS
THE NETWORK OF CENTERS IS BOTH
AN OPPORTUNITY TO COLLECT
BIOMARKER DATA AND TO IMPLEMENT
CLINICAL TRIALS.
I WAS JUST CURIOUS IF ANY
MEMBERS OF THE PANEL, BOB,
LAYNE, ANYBODY HAVE THOUGHTS
ABOUT THIS, WALTER AS WELL, HOW
YOU WOULD STREAMLINE HAVING A
CENTER THAT CAN DO THE MOST
IMPORTANT THINGS AND NOT
EVERYTHING WALTER WAS TALKING
ABOUT BECAUSE THERE'S A LOT OF
THINGS WALTER MENTIONED.
>>SO I'LL COMMENT BRIEFLY,
HANK.
I THINK THAT'S A GOOD ISSUE AND
IN FACT THAT WAS DISCUSSED IN
SOME OF THE MEETINGS.
SO FOR EXAMPLE, YOU WILL SEE IN
THE DOCUMENT THAT THERE ARE FOUR
TYPES OF CORES RECOMMENDED,
GENETICS IS ONE OF THEM.
SO I WOULD ARGUE THAT NOT EVERY
CENTER FOR EXCELLENCE SHOULD DO
EVERYTHING.
THAT IS TO SAY, NOT EVERYONE
SHOULD BE EXPECTED TO HAVE A
GENETICS CORE.
BUT I THINK WHAT SHOULD BE
MANDATORY IS AN INCLUSIVE REALLY
DIVERSE POPULATION THAT IS
CLINICALLY ASSESSED ACCORDING TO
STANDARDIZED APPROACHES WITH
QUANTITATIVE PHENOTYPIC FEATURES
THAT AT LEAST ALLOW CLINICAL
COMPARISON ACROSS MULTIPLE SITES
AND THAT PART OF THAT BE THE
COLLECTION FOR THE VARIOUS
REPOSITORIES OF BAYOU FLUIDS.
DNA AND SO FORTH.
I DON'T KNOW IF THAT ANSWERS
YOUR QUESTION, BUT I DON'T THINK
PERSONALLY THAT THEY NEED TO BE
CARBON COPIES OF EACH OTHER, BUT
I THINK THE CORE ELEMENTS DO
NEED TO ALLOW IN A MANDATORY WAY
SHARING ACROSS THE MULTIPLE
SITES.
AND PERHAPS EVEN WITH SITES
OTHER THAN FORMAL CENTERS OF
EXCELLENCE.
DOES THAT ADDRESS YOUR QUESTION,
HANK?
>>IT DOES, THANK YOU.
THE ONE THING I WILL SAY IS
THAT, FOR EXAMPLE, IN THE
ALZHEIMER'S DISEASE RESEARCH
CENTER NETWORK, THERE'S A DESIRE
THAT THOSE PLACES ARE ALSO DOING
CLINICAL TRIALS, BUT TO ME, IT
SEEMS ALMOST -- BASED ON WHAT I
HEARD, THAT THE NETWORK NEEDS TO
BE DOING CLINICAL TRIALS.
AND I DON'T KNOW HOW YOU BAKE
THAT IN.
YOU TALKED ABOUT A GENETICS
CORE.
THERE PROBABLY NEEDS TO BE A
CLINICAL TRIALS CORE THAT'S PART
OF THESE CENTERS AS WELL.
MAYBE NOT ALL OF THEM, BUT MOST
OF THEM.
>>I AGREE WITH THAT.
AND I THINK THAT WAS PART OF THE
CONCEPT BEHIND THE CLINICAL
CORE, BUT I THINK YOU'RE EXACTLY
RIGHT.
KEN?
>>YOU'RE ON MUTE, KEN.
>>I THINK KEN IS HAVING TROUBLE
UNMUTING.
WHY DON'T WE GO TO DAVID AND
WE'LL COME BACK TO YOU, KEN.
>>HI, EVERYBODY.
FIRST OF ALL, CONGRATULATIONS.
IT'S REALLY AN EXCITING SERIES
OF STUDIES THAT'S BEEN GOING ON.
WE ALSO SAW SOME TREMENDOUS WORK
ON A.L.S. PRESENTED YESTERDAY AT
THE INNOVATIVE TRIALS DESIGN FOR
TRAUMATIC BRAIN INJURY, A.L.S.
IS SOMETHING OF AN INSPIRATION
TO US IN THE FIELD OF TRAUMATIC
BRAIN INJURY.
ANOTHER INSPIRATION HAS BEEN THE
RECENT WORK ON BLOOD BRAIN
BARRIER TRAN CYTOSIS AND BLOOD
SPINAL CORD TRAN CYTOSIS.
SO IN ADDITION TO THE NEW TRIAL
MARKER TRIAL DESIGN CENTERS, I
REALLY THINK A KEY TECHNOLOGY
WITH GREAT POTENTIAL ADVANCE IN
A.L.S. TREATMENTS IN HUMANS
COULD BE THIS RECEPTOR MEDIATED
SHUTTLE TECHNOLOGY FOR
DELIVERING BIOLOGICAL MACRO
MOLECULES SUCH AS ANTIBODIES
ACROSS THE BLOOD BRAIN BARRIER,
THE WORK THAT WE SAW AT THE MOST
RECENT KEYSTONE DRUG DELIVERY TO
THE BRAIN KEYSTONE CONFERENCE
WAS REALLY REVOLUTIONARY,
THERE'S NOT MUCH WORK IN A.L.S.
AS OF YET, IT'S BEEN MOSTLY
ALZHEIMER'S DISEASE, BRAIN
CANCERS AND LYSOSOMAL STORAGE
DISEASING BUT I THINK IT HAS
TREMENDOUS POTENTIAL.
>>I AGREE.
I KNOW -- RITA, DO YOU WANT TO
COMMENT?
I FULLY AGREE.
>>NO, I ABSOLUTELY A.
THERE ARE AKNOW A COUPLE OF
GROUPS THAT STARTED WORKING IN
THIS FIELD AND I GREE THERE NEED
TO BE MORE EFFORT INTO REALLY
FIGURING THIS OUT AND HOW WE
TAKE ADVANTAGE OF THIS.
THANK YOU FOR POINTING THAT OUT.
>>KEN, ARE YOU ABLE TO --
>>SORRY ABOUT THAT.
I WAS HAVING SOME TECHNICAL
DIFFICULTIES.
SO I WAS LOOKING AT THE SORT OF
COMMENTS FROM A COUPLE OF OUR
COUNCILMEMBERS, AMY, BRANDON,
KRISTEN ABOUT THE GREAT EXTENT
AND DEGREE OF COLLABORATION
THAT'S GONE INTO THE STRATEGIC
APPROACH.
YOU KNOW, WHAT REALLY STUNNED
ME, AND WHAT I THINK MAKES THIS
VERY UNIQUE, AND PROBABLY WILL
LEAD TO A LOT OF SUCCESS IN THE
END, IS THAT, YOU KNOW, IN THE
PHARMACEUTICAL INDUSTRY, AND I
DON'T WANT TO TALK FOR ALL OF
PHARMACEUTICAL INDUSTRY, BUT I
FEEL THAT, YOU KNOW, I'M KIND OF
LIKE IN PART THE VOICE ON THE
COUNCIL HERE.
THERE'S A LOT OF EMPHASIS ON
PATIENT ENGAGEMENT.
AND EVERYONE IS DOING IT.
BUT WHAT I HEARD HERE GOES
BEYOND, YOU KNOW, MAKING SURE
YOU HAVE DIVERSE PATIENTS IN
YOUR CLINICAL TRIALS.
LIKE WE FOUND OUT WE STILL
DIDN'T DO IN THE COVID PANDEMIC,
OR EVEN HAVING PATIENTS
CONTRIBUTE TO YOUR CLINICAL
TRIAL PROTOCOL.
WHAT WE'RE HEARING HERE AND WHAT
WE'VE HEARD IS THAT THE
STRATEGIC INTENT AROUND THIS
DISEASE HAS BEEN DRIVEN BY THE
PATIENTS.
SO THERE ARE OBVIOUS CASES WHERE
LIKE HIGH PROFILE PATIENTS, LIKE
CHRISTOPHER REEVE AND SPINAL
CORD INJURY AND MICHAEL J. FOX
AND PARKINSON'S DISEASE, BUT
IT'S A COMPLETELY DIFFERENT
THING WHEN YOU'VE GOT REALLY
THOUGHTFUL, HIGH POWERED
INDIVIDUALS WHO ARE PATIENTS
THINKING AROUND THE STRATEGY OF
THE DISEASE AND HOW YOU GO
FORWARD IN THE R & D.
AND I THINK THAT THAT ALONE WILL
BRING PHARMA TO THE TABLE.
BECAUSE LET'S FACE IT, AND I
MADE THESE COMMENTS I THINK IN
THE LAST COUNCIL MEETING,
THERE'S NOT A LOT TRULY BIG
PHARMA IN THE A.L.S. SPACE.
RIGHT?
WE HAVE ESSENTIALLY WHAT, TWO
DRUGS, RILOTAC AND ADDARVON, AND
CLEARLY AAGREE WITH
DR. KOROSHETZ THAT IN ORDER FOR
THIS TO GO FORWARD, BIG PHARMA
HAS TO BE AT THE TABLE.
WHAT I'M SIGNALING TO YOU IS
THAT THE WAY THAT YOU'VE IMON
ABGONEABOUT DOING THIS, THE LEV-
THIS IS PATIENT ENGAGEMENT ON
STEROIDS.
THE LEVEL AT WHICH YOU'VE
BROUGHT THAT TO BEAR ON THIS
PROBLEM, I THINK IS A HUGE DRAW
FOR BIG PHARMA.
>>KEN, CAN I COMMENT ON THAT?
DO YOU MIND?
NOT EVERY PHARMA AND BIOTECH
COMPANY IS ON BOARD.
MOST ARE.
THE GOOD ONES ARE.
BUT YOU WOULD BE SURPRISED HOW
MANY ARE NOT ON BOARD REGARDING
PATIENT ADVOCACY AND ADVISORY
GROUPS.
THERE ARE MANY THAT ARE NOT.
BUT IT IS GROWING, THEY DO SEE A
NEED, THEY DO SEE VALUE, AND WE
ARE STRIVING AS A COMMUNITY, AS
A GROUP OF A.L.S. EXPERIENCE
EXPERTS, WE'RE ALWAYS
QUESTIONING PHARMA AND BIOTECH
TO ENSURE WE HAVE A SEAT AT THE
TABLE.
WE'RE TRYING AS HARD AS WE CAN.
AND AS I SAID, I COMMEND NIH AND
NINDS FOR DOING THIS.
IT SETS A GREAT PRECEDENT AND
HIGH BAR FOR ALL COMPANIES TO
HAVE US INVOLVED IN THE PROCESS
FROM THE BEGINNING.
>>TOTALLY AGREE.
>>THAT'S RIGHT.
THIS MIGHT BE A GOOD PLACE TO
STOP, ALTHOUGH THIS IS JUST THE
BEGINNING OF THE PROCESS.
I FOUND THIS INCREDIBLY
INSPIRING, AND IT'S NOT JUST
INSPIRING FOR A.L.S., WHICH IT'S
OBVIOUSLY INSPIRING FOR, BUT FOR
MANY OF THE OTHER DISEASES THAT
WE'RE RESPONSIBLE FOR, THIS
APPROACH BEING TAKEN HERE.
SO WE'LL BE HEARING MORE.
THANKS A MILLION TO ALL OF YOU
WHWHO PARTICIPATED IN THIS.
IT'S REALLY BEEN GREAT.
OKAY.
IT'S HARD TO SHIFT GEARS NOW.
BUT I'M GOING TO GO ON TO THE
LAST SECTION OF THE MEETING,
WHICH HAS TO DO WITH CONCEPT
CLEARANCE.
THERE'S ACTUALLY AN A.L.S.
CONCEPT INCLUDED IN THAT.
AND BE BEFORE WE LAUNCH ANY NEW
INITIATIVE AT NINDS, WE ASK FOR
CLEARANCE FROM COUNCIL.
WE'VE BEEN DOING THIS FOR A
LONG, LONG TIME.
BUT IT'S ACTUALLY NOW REQUIRED
BY NIH.
SO MOST OF THE INITIATIVES THAT
WE NEED CLEARANCE FOR ARE
ACTUALLY REISSUES THAT WE'RE NOT
GOING TO TALK ABOUT.
I'M GOING TO SHOW A SLIDE WITH
THEM AND IF YOU HAVE QUESTIONS,
WE CAN TALK ABOUT THEM, BUT
THERE ARE A FEW THAT H WE ARE
GOING TO DISCUSS.
THE ONE WE'LL SPEND THE MOST
TIME IS THE VERY FIRST ONE,
WHICH IS -- IT'S A PROPOSED
PROGRAM, AND BY THE WAY, THIS
HAS IMPLICATIONS FOR ALL THE
DISEASES THAT WE COVER, AS WELL
AS BASIC RESEARCH.
SO THE FIRST IS THIS PROPOSED
PROGRAM TO ENHANCE SCIENTIFIC
RIGOR IN NEUROSCIENCE
DEPARTMENTS ACROSS THE COUNTRY.
VERY INNOVATIVE, INTERESTING
PROGRAM.
DEVON, WE BLOCKED OFF SOME TIME
FOR THIS ONE MORE THAN WE
USUALLY SPEND BECAUSE IT'S SO
NEW AND WE REALLY DO WANT TO
DISCUSS IT.
THIS IS NOT JUST GOING TO BE A
PRESENTATION.
DEVON CRAWFORD, WHO'S IN OUR
NINDS OFFICE OF RESEARCH
QUALITY, WHICH IS HEADED BY
SHI -- IS DEVON THERE?
I'M LOOKING FOR HER.
I SEE YOUR SLIDES, DEVON.
I'M NOT SURE I SEE YOU.
>>OKAY.
HOPEFULLY YOU CAN SEE ME NOW.
>>THERE YOU ARE.
OKAY.
TAKE IT AWAY.
>>ALL RIGHT.
LET ME JUST MOVE THIS.
OF ALL RIGHT.
>>THANK YOU, BOB.
HOPEFULLY EVERYBODY IS HANGING
IN THERE THIS AFTERNOON.
I'M HAPPY TO BE HERE TODAY
SPEAKING ON BEHALF OF THE ENTIER
NINDS OFFICE OF RESEARCH QUALITY
IN ORDER TO SOLICIT YOUR
FEEDBACK ON THIS IDEA FOR A NEW
PROGRAM, AND THIS IS A PROPOSAL
FOR A NEW NINDS FUNDING
OPPORTUNITY TO CREATE
SUSTAINABLE TRANSFORMATION OF
INSTITUTIONAL RESEARCH RIGOR IN
THE NEUROSCIENCES.
AND WITH THIS, WE HOPE TO
STIMULATE A STRONGER CULTURE OF
PERFORMING AND REWARDING HIGH
QUALITY RESEARCH IN A WAY THAT
WILL COMPLEMENT OUR OTHER
EFFORTS IN THIS SPACE.
SO WE LOOK FORWARD TO YOUR
FEEDBACK.
SO WHY IS EXPERIMENTAL RIGOR OUR
FOCUS?
AS OUR OWN DIRECTOR DR. WALTER
KOROSHETZ WROTE IN HIS 2021
DIRECTOR'S MESSAGE, GOOD SCIENCE
IS RIGOROUS SCIENCE.
SO IN OTHER WORDS, HIGH QUALITY
RIGOROUS RESEARCH IS
FOUNDATIONAL TO THE SCIENTIFIC
ENTERPRISE.
IN THE LATEST STRATEGIC PLAN AS
CROSS-CUTTING STRATEGIES,
INTEGRATED ACROSS INSTITUTE
ACTIVITIES.
AND IN THIS PLAN, NINDS ACTUALLY
ACKNOWLEDGED THAT RESEARCH IS
OFTEN FLAWED OR NOT
TRANSPARENTLY REPORTED, WHICH
CAN UNDERMINE SCIENTIFIC
ADVANCEMENTMENT.
ADVANCEMENT.
FOR EXAMPLE, FLAWS IN ACADEMIC
PRE-CLINICAL STUDIES, THEY
SHOWED THAT THEY COULD NOT
REPLICATE ABOUT 65% OF EARLY
TARGET IDENTIFICATION AND
VALIDATION PROJECTS THAT THEY
PERFORMED IN HOUSE.
AND THIS ACTUALLY RAISED AN
ALARM AND OTHER GROUPS FOUND
SIMILAR ISSUES WITH PRE-CLINICAL
REPRODUCIBILITY IN SUBSEQUENT
STUDIES.
AND THIS MAY NOT SEEM LIKE A
MAJOR ISSUE SINCE WE WOULDN'T
EXPECT EVERY NEW FINDING TO BE
PERFECTLY REPRODUCIBLE, BUT IT
CAN HAVE NEGATIVE CONSEQUENCES
FOR HUMAN HEALTH AND WELL-BEING,
ESPECIALLY IF MUCH OF THE
IRREPRODUCIBILITY IS AVOIDABLE.
AND THIS ISSUE IS HIGHLIGHTED BY
STUDIES ON THE SOD1 MOUSE MODEL
OF A.L.S., SO THIS IS PROBABLY
VERY RELEVANT TO THE DISCUSSION
WE JUST HAD.
IN THE EARLY 2000s, MANY DRUGS
HAD BEEN FOUND TO IMPROVE
SURVIVAL IN THIS MOUSE MODEL
BRINGING HOPE OF TREATMENTS TO
PEOPLE WITH ALZHEIMER'S.
AND YOU CAN SEE THE MEAN
SURVIVAL RATE IN THESE MICE FOR
EACH OF THESE DRUGS IN THESE
LIGHT BLUE BARS THAT GO OFF TO
THE RIGHT.
HOWEVER, ALMOST EVERY SINGLE ONE
OF THESE DRUGS FAILED COMPLETELY
IN EXPENSIVE, TIME-CONSUMING
HUMAN CLINICAL TRIALS, BUT AFTER
THESE FAILURES, THE A.L.S.
THERAPY DEVELOPMENT INSTITUTE
ACTUALLY REPEATED THE
PRE-CLINICAL WORK AND FOUND THAT
EACH OF THESE DRUGS DID NOT
ACTUALLY IMPROVE SURVIVAL IN
MICE AND YOU CAN SEE THE MEAN
SURVIVAL THAT THEY FOUND IN THE
DARK BARS.
SO WHAT WAS DIFFERENT THIS TIME
AROUND?
WELL, THE NEW EXPERIMENTS THEY
PERFORMED HAD BLINDING OF
INVESTIGATORS.
THEY RANDOMIZED MICE TO
TREATMENT GROUP.
THEY HAD SUFFICIENT SAMPLE SIZE
THAT THEY CHOSE IN ADVANCE.
THEY MATCHED FOR SEX AND LITTER.
THEY PREDEFINED THEIR INCLUSION
AND EXCLUSION CRITERIA TO REDUCE
CONFOUNDING FACTORS AND THEY
TRANSPARENTLY REPORTED THAT THEY
DID EACH OF THESE ITEMS.
AND SO IN SHORT, WHEN THESE
MOUSE STUDIES WERE PERFORMED
MORE RIGOROUSLY, THEY BETTER
MATCHED THE RESULTS SEEN IN
CLINICAL TRIAL.
SO I OFTEN IMAGINE HOW MUCH
TIME, MONEY AND PATIENT RISK
COULD HAVE BEEN AVOIDED IF THE
ORIGINAL ANIMAL STUDIES HAD JUST
BEEN PERFORMED MORE RIGOROUSLY.
NOW, THE QUESTION IS WHY ARE
PRE-CLINICAL STUDIES OFTEN NOT
AS RIGOROUS OR AS TRANSPARENT AS
THEY COULD BE?
AND I DON'T ENTEARLY BLAME
SCIENTISTS BECAUSE THEY'RE NOT
PERFORMING THE WORK IN A VACUUM.
THEY'RE UNDER VARIOUS PRESSURES.
THERE ARE PROFESSIONAL SOCIETIES
THAT DEVELOP FIELD-SPECIFIC
STANDARDS AND CAREER DEVELOPMENT
EXPECTATIONS FOR SCIENTIST.
SCIENTISTS.
JOURNALS OFTEN PIT SCIENTISTS
AGAINST EACH OTHER TO TELL THE
FLASHIEST AND MOST EXCITING
STORY POSSIBLE IN ORDER TO EVEN
GET PUBLISHED.
YOU HAVE FUNDERS AND PEER
REVIEWERS THERE THAT OFTEN TAKE
PUBLICATION PRODUCTIVITY INTO
ACCOUNT WHEN EVEN CONSIDERING
THAT INVESTIGATOR'S EXPERTISE
BUT THEN ON TOP OF IT ALL,
INSTITUTIONAL PROMOTION AND
TENURE COMMITTEES RELY HEAVILY
ON PUBLICATION RECORD AND
FUNDING HISTORY BH DECIDING WHEG
ABOUT CAREER ADVANCEMENT.
CAREFUL SCIENCE CAN BE
MISCONSTRUED AS TOO SLOW OR
BORING TO GARNER THE FLASHY
PUBLICATIONS AND THE ABUNDANT
GRANT FUNDING EVEN THOUGH IT MAY
BE MORE LIKELY TO BE
SCIENTIFICALLY VALID.
AND SO ALL OF THESE PARTIES HAVE
A RESPONSIBILITY FOR FOSTERING A
CULTURE THAT ACTUALLY
INCENTIVIZES RIGOR AND
TRANSPARENCY AND ALL OF THEM
NEED TO ADAPT IF WE WANT TO SEE
A CHANGE IN THAT CULTURE.
FOR THIS REASON, NINDS HAS
ALREADY STARTED TAKING AN ACTIVE
ROLE IN TRYING TO CHANGE THE
CULTURE OF SCIENCE BY
INFLUENCING EACH OF THESE
CONTRIBUTORS TO THE ECOSYSTEM.
FOR EXAMPLE, FOR PUBLISHERS, WE
WORK TO INCREASE TRANSPARENCY BY
DEFINING A MINIMAL SET OF ITEMS
TO REPORT FOR EVERY STUDY OFTEN
REFERRED TO AS THE LANDIS
4 AFTER A WORKSHOP SUMMARY THAT
WE PUBLISHED.
WE ALSO BROUGHT A PROPOSAL TO
LAST COUNCIL FOR REDUCING
PUBLICATION BIAS AGAINST NO
RESULTS THROUGH NIH
INFRASTRUCTURE AND INCENTIVE AS
A FUNDER.
YOU MIGHT ALREADY BE AWARE OF
THE CURRENT NIH-WIDE GRANT
REVIEW CRITERIA THAT INCORPORATE
FOUR AREAS OF FOCUS RELATED TO
RIGOR BUT YOU MIGHT NOT BE AWARE
OF SOME OF OUR LOCAL ACTIVITIES
HERE AT NINDS.
WE HAVE PUBLISHED GUIDANCE ON
OUR WEBSITE ABOUT RIGOR ELEMENTS
TO CONSIDER WHEN WRITING NIH
GRANT APPLICATIONS.
IN THE LAST YEAR WE'VE ALSO MADE
A CONSCIOUS EFFORT TO INCREASE
ATTENTION TO RIGOR AND
TRANSPARENCY IN FUNDING
OPPORTUNITY ANNOUNCEMENTS,
THAT'S INCLUDING THE FOA-SMEF
REVIEW CRITERIA, AND WE'VE ALSO
MADE IT CLEAR ON OUR WEBSITE
THAT WE CONSIDER AN
INVESTIGATOR'S RIGOR AND
TRANSPARENCY WHEN DECIDING
WHETHER OR NOT TO FUND A GRANT
THROUGH SELECT PAY.
SO THAT'S YOUR HIGH PROGRAM
PRIORITY OR BRIDGE AWARDS.
WE'VE ALSO WORKED WITH
SCIENTIFIC SOCIETIES TO CRAFT
GUIDANCE FOR IMPROVING
TRANSPARENCY IN CONFERENCE TALKS
IN POSTER PRESENTATIONS, AND ON
THE INDIVIDUAL LEVEL, WE ALSO
PARTICIPATE IN COMMUNITY
ENGAGEMENT THROUGH TALKS, SOCIAL
MEDIA, WORKSHOPS.
WE HAVE ONLINE RESOURCES.
YOU MAY HAVE ALSO HEARD AT A
PREVIOUS COUNCIL MEETING THAT WE
ARE DEVELOPING A LARGE ONLINE
EDUCATIONAL RESOURCE TO IMPROVE
TRAINING IN THE PRINCIPLES OF
RIGOROUS RESEARCH, AND WE ARE
ALSO PROPOSING A NEW CHALLENGE
PRIZE.
THE PURPOSE OF WHICH IS TO
IDENTIFY AND REWARD INDIVIDUALS
OR SMALL GROUPS FOR INCREASING
ATTENTION, RIGOR AND
TRANSPARENCY ABOVE AND BEYOND
THEIR NORMAL JOB DUTIES.
HOWEVER, YOU MIGHT NOTICE ONE
CULTURAL KEYSTONE MISSING FROM
THIS LIST AND THAT'S
INSTITUTIONS.
NOW WHAT DOES NINDS AFFECTING
LOCAL INSTITUTIONAL CULTURE?
WELL, NOT THAT MUCH.
NOW, NIH POLICIES, OF COURSE,
WILL GUIDE INSTITUTIONAL
BEHAVIOR THROUGH MANDATES LIKE
THE DATA SHARING OR TRAINING
REQUIREMENTS, BUT NINDS HAS NOT
YET TRIED TO DIRECTLY
INCENTIVIZE INSTITUTIONS TO
PROMOTE AND ENHANCE RIGOR VIA
THE LOCAL RESEARCH CULTURE.
SO FOR THIS REASON, WE ARE
PROPOSING THE NINDS SUSTAINABLE
TRANSFORMATION OF INSTITUTIONAL
RESEARCH RIGOR, OR STIRR,
PROGRAM, AND THIS INITIATIVE
WILL SUPPORT THE ESTABLISHMENT
OF PROGRAMS TO ENHANCE RESEARCH
RIGOR AND TRANSPARENCY PRACTICES
WITHIN ACADEMIC AND RESEARCH
INSTITUTIONS IN ORDER TO PROMOTE
A CULTURE OF HIGH QUALITY
NEUROSCIENCE RESEARCH.
SO THIS FUNDING OPPORTUNITY IS
MEANT TO SUPPORT AN INDIVIDUAL
DEPARTMENT, OFFICE, OR OTHER
INTRA-INSTITUTIONAL ENTITY.
SO THIS IS NOT THE LEVEL OF THE
ENTIRE INSTITUTION BUT RATHER AT
A MORE LOCAL LEVEL WITHIN THE
INSTITUTION.
AND SINCE WE'RE NINDS, WE WILL
ALSO FOCUS ON ENTITYS THAT HAVE
A CRITICAL MASS OF
NEUROSCIENTISTS THAT WILL
BENEFIT FROM THE PROGRAM, SO FOR
EXAMPLE, YOU KNOW, AT LEAST SOME
OF THE LABS WITHIN A DEPARTMENT
WOULD NEED TO BE PERFORMING
RESEARCH IN THE NINDS MISSION
SPACE FOR THAT DEPARTMENT TO BE
ELIGIBLE TO APPLY.
AND FINALLY WE ARE PROPOSING A
THREE-YEAR LIMIT TO THE AWARD
BECAUSE THIS SHOULD BE
KICK-STARTING A NEW PROGRAM
RATHER THAN SUSTAINING ALREADY
SUCCESSFUL PROGRAMS.
AND THESE ARE FEATURES THAT WE
ENVISION SHOULD BE INTEGRATED
INTO THE APPLICANT PROGRAMS.
WE WANT TO SEE CREATIVE
SOLUTIONS TO GAPS THAT WERE
IDENTIFIED BY THE INSTITUTIONAL
ENTITY.
WE WANT THERE TO BE HYPOTENSIONH
POTENTIAL IMPACT FOR IMPROVING
RIGOR AND TRANSPARENCY.
WE WANT TO SEE THEM INTEGRATING
THESE NEW PRACTICES INTO THEIR
EVERYDAY CULTURE AS WELL AS, OF
COURSE, EVALUATING THE PROGRAM
AND DISSEMINATING THOSE RESULTS.
WE ALSO WANT TO SEE STRONG
INSTITUTIONAL COMMITMENT TO
SUSTAINABILITY BEYOND THE AWARD,
AS WELL AS SOME POTENTIAL FOR
SCALABILITY BEYOND THE ENTITY.
BECAUSE ULTIMATELY, WE WANT TO
IDENTIFY SUCCESSFUL PROGRAMS
THAT COULD SPREAD TO OTHER
ENTITIES INSIDE OR OUTSIDE OF
THE AWARDEE INSTITUTION.
AND WE ENVISION THAT THIS WILL
SUPPORT A VARIETY OF TYPES OF
PROGRAMS UNDER THIS INITIATIVE,
RANGING FROM MORE FOCUS TO
BROADER ACTIVITIES.
FOR EXAMPLE, WE COULD SEE
APPLICANTS PROPOSED TO IMPLEMENT
RECOGNITION AND AWARDS TO
PROMOTE AWARENESS OF AND
INCENTIVIZE BEST PRACTICES.
WE COULD SEE DEDICATED
SPECIALISTS LIKE DATA MANAGERS
OR RESEARCH METHODOLOGISTS TO
HELP THEM CHANGE THEIR EVERYDAY
LABORATORY WORKFLOWS.
WE COULD SEE RESTRUCTURING
TRAINEE PROGRAMS TO EMPHASIZE
THE QUALITY OF THE RESEARCH OVER
SAY A CERTAIN NUMBER OF
PUBLICATIONS BEING REQUIRED FOR
GRADUATION.
THERE COULD BE A RESTRUCTURING
OF HIRING PROMOTION AND TENURE
CRITERIA TO AGAIN EMPHASIZE
RIGOR AND TRANSPARENCY OVER
THINGS LIKE SAY TAITION COUNT OR
JOURNAL IMPACT FACTOR.
THERE COULD BE SOME SORT OF
POLICIES IN PLACE, THIS COULD BE
THAT ALL PUBLICATIONS NEED TO
HAVE A MINIMAL REPORTING ITEM IN
ORDER TO GO OUT THE DOOR, THERE
COULD BE S.O.P.s AND
INCORPORATION OF NEW
INFRASTRUCTURE.
THIS COULD BE SOCIALIZING
ELECTRONIC LAB NOTEBOOKS OR
IMPLEMENTING QUALITY MANAGEMENT
SYSTEMS.
THERE COULD BE COMPREHENSIVE
EDUCATIONAL OR PROFESSIONAL
DEVELOPMENT PROGRAMS, OR
FINALLY, PARTNERSHIPS WITH
OUTSIDE ORGANIZATIONS IN ORDER
TO TRANSFORM HOW THE ENTITY
OPERATES.
AND SO ALTHOUGH VERY DIFFERENT
PROGRAMS, EACH OF THESE EXAMPLES
WOULD NEED TO BE JUSTIFIED AND
FEASIBLE FOR THE PARTICULAR
ENTITY APPLYING.
SO IN SUMMARY, WE'D LIKE THERE
TO BE FEWER DEMANDS ON
SCIENTISTS THAT DISINCENTIVIZE
RIGOROUS AND TRANSPARENT
RESEARCH PRACTICES, AND WITH
THIS PROPOSED PROGRAM, WE HOPE
TO SEE ENTITIES WITHIN
INSTITUTIONS DOING THEIR PART TO
SUPPORT RESEARCHERS TO BETTER
VALUE RIGOR AND TRANS PAREN
SEAL, AND CHANGE HOW RESEARCH IS
ASSESSED AND REWARDED,
SUPPORTED, PRODUCED EVEN,
THEREBY REVERSING THE PRESSURE
ON SCIENTISTS, AND OUR WORKING
HYPOTHESIS IS THAT IF SCIENTISTS
RECEIVE THIS ADEQUATE SUPPORT
AND THEY SEE THIS ALIGNMENT OF
VALUES AND INCENTIVES FROM THEIR
DEPARTMENTS, THEY MAY FEEL MORE
EMPOWERED TO BOLSTER THEIR OWN
RESEARCH PRACTICES, AND
TRANSFORM A LOCAL INSTITUTIONAL
CULTURE, WHICH ITSELF WOULD
ENABLE ANY CHANGES TO BECOME
SUSTAINABLE.
AND SO WE FEEL THAT THIS PIECE
IS MISSING FROM OUR CURRENT
PORTFOLIO OF INITIATIVES THAT
ARE OUT THERE TO ENHANCE
TRANSPARENCY.
SO WITH THAT, I WILL HO OPEN ITP
FOR QUESTIONS AND FEEDBACK.
THANK YOU.
AND I'LL STOP SHARING.
>>THANKS A LOT, DEVOFNL THE ONE
THING YOU CAN SEE IS THIS IS A
VERY AMBITIOUS PROGRAM.
THEY'RE REALLY TRYING TO TACKLE
AN INKR INCREDIBLY IMPORTANT AND
INCREDIBLY DIFFICULT ISSUE.
AND THIS IS ALSO A PRETTY BRAND
NEW PROPOSAL.
SO WE REALLY WOULD LIKE TO HEAR
WHAT YOU THINK, IF YOU HAVE
IDEAS THAT COULD BE POTENTIALLY
INCORPORATED INTO THIS, IF YOU
THINK THIS IS THE RIGHT
APPROACH, ET CETERA.
SO I'LL THROW OPEN THE FLOOR.
BECAUSE MANY OF YOU KNOW BETTER
THAN WE DO, YOU KNOW, HOW YOUR
INSTITUTIONS CHANGE.
LET'S START WITH SAMEER, THEN
HANK GREELY.
>>THANKS, DEVON.
VERY INTERESTING, LIKE BOB SAID,
SUPER AMBITIOUS.
AND YOU'RE FIGHTING A BATTLE ON
MANY FRONTS.
YOU KNOW, I WONDER IF -- SO I
DON'T HAVE A SOLUTION TO ANY OF
THAT, BUT I WONDER IF THERE'S
ANOTHER PART OF THIS THAT CAN BE
INCORPORATED, MAYBE NOT TO MAKE
IT MORE COMPLICATED BUT MAYBE TO
TRY TO GET MORE BANG FOR YOUR
BUCK, WHICH IS JUST SO MUCH THAT
WE HAVE TALKED ABOUT ON DATA
SHARING.
THERE'S A LOT OF, I THINK,
OVERLAP BETWEEN RIGOR,
REPRODUCIBILITY AND SHARING OF
DATA, AND OBVIOUSLY THE LATTER
IS BEING MANDATED AT THE VARIOUS
LEVELS, WE'RE ALREADY DOING SOME
OF IT, WE NEED TO DO MORE OF IT
AS OF LIKE FIVE DAYS AGO, RIGHT?
AND NOT ONLY WOULD ALIGNING
THESE KIND OF EFFORTS ALLOW FOR
DATA AND ANALYSES TO BE CHECKED
IN TERMS OF RIGOR, BUT IT WOULD
ALSO OBVIOUSLY ALLOW GROUPS TO
BE ABLE TO USE DATA MORE
EFFECTIVELY, WHICH IS NOT
NECESSARILY PART OF THE SBIR,
BUT IT'S ANOTHER ASPECT OF
INITIATIVES THAT WE'RE
INTERESTED -- BY BRAIN AND
NINDS, ET CETERA.
SO I WONDER IF THERE'S SOME
OVERLAP THERE WHERE PART OF THIS
INITIATIVE ALSO FOCUSES ON BEST
PRACTICES FOR DATA SHARING.
THERE'S A LOT OF OVERLAPPING
INCENTIVES OR DISINCENTIVES, YOU
KNOW, WHEN YOU TALK ABOUT DATA
SHARING, IF YOU'RE THE ONE
SHARING DATA, WHAT DO YOU GET
OUT OF IT?
IF YOU'RE USING SHARED DATA,
WHAT DO YOU LOSE AS IN JOURNALS
ARE NOT GOING TO BE AS EXCITED
ABOUT RECYCLED DATA.
SO THERE'S A LOT OF THAT SORT OF
INCENTIVE/DISINCENTIVE
CALCULATION THAT COMES IN WITH
DATA SHARING THAT I THINK
OVERLAPS WITH THOSE CALCULATIONS
FOR THIS INITIATIVE.
SO I WONDER IF, YOU KNOW, THAT
COULD BE SOMETHING THAT YOU'VE
THOUGHT ABOUT.
>>YEAH, SO YOU KNOW, I SEE SORT
OF DATA SHARING, OPEN SCIENCE,
AS PART OF THE TRANSPARENCY ARM,
AND SO THAT'S NOT THE ONLY THING
WE WOULD LIKE TO SEE COME IN
BECAUSE, OF COURSE, THERE ARE A
LOT OF EFFORTS IN THE DATA
SHARING SPACE CURRENTLY.
ODSS HAS A LOT OF INITIATIVES
GOING ON.
BUT ABSOL ABSOLUTELY, WE MENTION
THERE HAVING DATA MANAGERS,
HELPING PEOPLE ORGANIZE AND
CHANGE THEIR WORKFLOWS.
THAT'S ABSOLUTELY WITHIN SCOPE
OF WHAT WE'RE THINKING.
AND SO IT'S ABOUT LIKE CHANGING
THAT CULTURE AND TEACHING PEOPLE
HOWL TO DO THINGS BETTER, BUT
HOW TO MAKE THOSE CHANGES
SUSTAINABLE.
THAT'S REALLY WHAT WE WANT TO
SEE, IS THAT CULTURE CHANGE.
>>GOOD QUESTION.
HANK.
>>SO THIS SOUNDS REALLY
IMPORTANT, REALLY AMBITIOUS AND
REALLY HARD.
CHANGING ANYBODY'S CULTURE IS
HARD, CHANGING OTHER PEOPLE'S
CULTURE IS PROBABLY EVEN HARDER.
WHEN I THINK ABOUT WHAT NINDS
CAN DO TO CHANGE WHAT STANFORD
CONSIDERS THE APPROPRIATE STAGE
FOR TENURE, GOOD LUCK.
I'M NOT SURE THAT ANYBODY AT
STANFORD CAN CHANGE WHAT
STANFORD CONSIDERS THE
APPROPRIATE LEVEL FOR STANFORD.
BUT THERE ARE THINGS THAT NINDS
AND NIH MORE BROADLY HAVE SOME
CONTROL OVER.
I THINK YOU CAN GET A LOT OF
RESEARCHERS' ATTENTION IF, FOR
EXAMPLE, YOU FIGURED OUT A WAY
TO HAVE STUDY SECTIONS PAY
ATTENTION TO THE RIGOR OF
APPLICANTS' PAPERS.
AND THE RIGOR WITH WHICH THE
PAPERS THAT SUPPORT THE
PARTICULAR APPLICATION GO.
NOW THIS COULD BE PRESUMABLY
NICE, NON-CONTROVERSIAL GOOD
LUCK ON BOTH OF THOSE WAYS TO
MEASURE RIGOR, BUT THAT WOULD
GET PEOPLE'S ATTENTION, AND
THAT'S UNDER NIH OR NINDS
CONTROL.
>>TAKE IT AWAY, DEVON.
>>I MEAN, I WILL SAY THAT I
DON'T DISAGREE THAT THAT IS A
VERY INTERESTING IDEA.
AND THERE ARE ISSUES FOR EXAMPLE
THAT CURRENTLY -- WITH GRANT
APPLICATIONS THAT REVIEWERS ARE
NOT REQUIRED TO LOOK UP ANYTHING
THAT IS NOT PROVIDED WITH THE
GRANT APPLICATION, SO THAT WOULD
BE, YOU KNOW, A POLICY ISSUE WE
WOULD HAVE TO CONTEND WITH.
BUT ABSOLUTELY, AS PART OF OUR
SELECT PAY, SO THAT'S BEYOND THE
PAYLINE, IT'S AFTER THE
SCIENTIFIC MERIT REVIEW.
WE DO CONSIDER INVESTIGATOR
RIGOR WHEN LOOKING AT SELECT PAY
ITEMS.
SO THAT'S SOMETHING WE CAN DO
PROGRAMMATICALLY.
BUT YOU KNOW, CHANGING CENTRAL
CSR REVIEW CRITERIA TO ALLOW FOR
LOOKING THROUGH PEOPLE'S PAPERS,
THAT'S, YOU KNOW, NOT SOMETHING
I'M GOING TO COMMENT OR PROMISE
THAT WE CAN DO.
>>BUT IN A BROADER SENSE, HANK,
SHI AND OTHERS HAVE HAD AN
ENORMOUS EFFECT ON CSR'S SOPs
FOR DOING REVIEWS.
MAYBE NOT AS MUCH OF AN EFFECT
AS THEY WANTED BUT STUDY
SECTIONS ARE NOW TAKING -- THE
GOAL IS FOR STUDY SECTIONS TO
TAKE RIGOR MUCH MORE INTO
ACCOUNT THAN THEY DID IN THE
PAST.
THIS IS A DIFFICULT ASK, BUT
THEY HAVE -- SHI AND ALL HAVE
CHANGED THAT CULTURE.
BUT THERE'S STILL A WAY TO GO.
>>FOR EXAMPLE, IF WE ARE TAKING
INTO ACCOUNT RIGOR ON THE
SELECTED ONES BELOW THE
PAYLINE -- MAKING SURE THAT
APPLICANTS KNOW THIS IS --
[SPEAKER CUTTING OUT]
>>DID HE FREEZE FOR YOU ALL AS
WELL?
>>YES.
>> -- HERE I AM IN THE MIDDLE
OF SILICON VALLEY AND MY
INTERNET HAS BEEN ERRATIC AT
BEST DURING THIS.
I APOLOGIZE.
I WISH I KNEW HOW TO FIX IT.
ALL I WAS SAYING WAS, HAVE WE
PUBLICIZE -- TO THE EXTENT WE
TAKE RIGOR INTO CONSIDERATION,
IN THE ONES BETWEEN 14
PERCENTILE AND 25TH PERCENTILE
OR WHATEVER THE SELECT ONES ARE,
HOW MUCH DO APPLICANTS KNOW
THAT?
HOW MUCH -- IS THAT SOMETHING
THAT IS SIGNIFICANT ENOUGH OR
APPEARS TO APPLICANTS TO BE
SIGNIFICANT ENOUGH FOR POSSIBLY
TO CHANGE THEIR BEHAVIOR?
>>I WOULD SAY I DON'T KNOW WHAT
THE APPLICANT PERCEPTION IS, BUT
I DO KNOW WE HAVE IT ON OUR
WEBSITE BUT WE PROBABLY COULD DO
A BETTER JOB OF ADVERTISING
THAT.
>>HANK, FOR A GUY WHO PROFESSES
NOT TO UNDERSTAND NIH VERY WELL,
YOU UNDERSTAND NIH VERY WELL.
I THINK WE DO NEED TO PUBLICIZE
THAT MORE THAN WE CURRENTLY DO.
MANY APPLICANTS DON'T REALIZE
THAT IF THEY'RE IN THE SELECTIVE
PAY ZONE, THAT WE REALLY DO PAY
ATTENTION TO RIGOR.
SO I THINK THAT'S AN IMPORTANT
POINT.
>>I WILL ADD, I DON'T THINK I
UNDERSTAND NIH VERY WELL, BUT I
DON'T THINK ANYBODY UNDERSTANDS
NIH VERY WELL.
>>THERE USED TO BE A STORY BY
THOMAS WOLF CALLED "ONLY THE
DEAD KNOW BROOKLYN," WHICH IS A
VERY SIMILAR PRINCIPLE.
GINA.
>>I WANTED TO ACTUALLY SORT OF
PIGGY BACK ON HANK'S COMMENT AND
I'D SAY THAT I ACTUALLY BELIEVE
THERE IS NO FASTER WAY TO CHANGE
CULTURE REGARDING RIGOR AND
REPRODUCIBILITY OF SCIENCE THAN
NIH POLICY.
I THINK IF THE NIH CHANGES THEIR
POLICY AND MAKES IT SO THAT, YOU
KNOW, EVERYBODY HAS TO POST
THEIR DATA AND EVERYBODY HAS TO
PUBLISH ONE FIGURE AT A TIME AS
THE DATA COME OUT, YOU KNOW, AS
THE DATA ROLL OFF THE BENCH, I
THINK EVERY -- STANFORD
INCLUDED, EVERY DEPARTMENT THAT
RELIES ON NIH MONEY WILL SIT UP
AND TAKE NOTICE AND CHANGE THEIR
POLICY AND CHANGE THE WAY THAT
THEY PROMOTE.
I THINK REALLY THERE IS NO
FASTER WAY TO GET PUBLICATIONS
TO CHANGE THEIR POLICY THAN FOR
NIH TO SAY, LISTEN, YOU KNOW,
WE'RE NOT GOING TO FUND THIS
RESEARCH UNLESS THIS HAPPENS.
SO I THINK THIS IS EXTREMELY
EXCITING, I THINK IT WILL BE
EXTREMELY IMPACTFUL AND I WANT
TO EMPOWER YOU TO TAKE STRONG
MEASURES BECAUSE THIS IS THE WAY
TO DO IT, THIS IS THE BEST WAY
TO DO IT.
>>THANKS A LOT, GINA.
JOHN?
>>YEAH, I JUST REALLY THANK
YOU, DEVON, FOR THAT
PRESENTATION.
I REALLY JUST WANTED TO ADD MY
VOICE IN SUPPORT OF THIS EFFORT.
IT IS SO IMPORTANT, YOU KNOW,
ALL THE INCENTIVES ARE IN THE
WRONG PLACE RIGHT NOW, AND IT'S
COSTING US ENORMOUSLY IN TERMS
OF RESEARCH PRODUCTIVITY.
SO I'M REALLY LOOKING FORWARD TO
SEEING WHAT COMES OUT OF THIS.
I HOPE THERE WILL BE SOME WAY IN
WHICH WE CAN GET THE
RELIABILITY, THE RIGOR OF THE
WORK IN AN ARTICLE SOMEHOW MORE
VISIBLE AND OBVIOUS AND
CONSPICUOUS AS PEOPLE ARE
READING THE LITERATURE.
BECAUSE I THINK IT'S SO
IMPORTANT FOR US THAT THAT
MEASURE GET UP FRONT.
SO THANK YOU FOR YOUR WORK ON
THIS.
>>THANKS, JOHN.
DAVID?
>>COULDN'T AGREE MORE WITH WHAT
EVERYONE HAS SAID SO FAR.
WITH JOURNAL, WE'RE REQUIRING
THE STATEMENT WITH EVERY
MANUSCRIPT STARTING JANUARY 1ST
AND WE'LL SEE WHAT SORT OF
EFFECT THAT HAS ON OUR FIELD.
WE ALSO HAVE A SIMILAR
REPRODUCIBILITY CRISIS IN THE
FIELD OF TRAUMATIC BRAIN INJURY.
AND AS PER YOUR POINT,
DR. CRAWFORD, ABOUT WHAT THE
APPLICANTS THINK, THEY MOSTLY
THINK ABOUT GOSH IF I'M
PUBLISHED IN A HIGH PROFILE
JOURNAL, I HAVE A GOOD CHANCE OF
GETTING MY GRANT FUNDED.
KIND OF THE GENERAL RULE THAT
APPLICANTS OFTEN THINK IS ONE
HIGH PROFILE PAPER AS
PRELIMINARY DATA PER AIM.
AND IF YOU'RE GOOD THERE, YOU'RE
GOOD TO GO.
SO THAT'S KIND OF LIKE, I DON'T
KNOW, MY FEELING ABOUT THE
GENERAL GESTALT OUT THERE.
OVER.
>>THANKS, DAVID.
TIM?
>>I, OF COURSE, SECOND
EVERYBODY'S OPINION.
I THINK THAT ONE OF THE
CHALLENGES I'VE SEEN AS BOTH A
REVIEWER AND WRITER OF GRANTS
WHERE YOU HAVE TO PUT IN
STATEMENTS IS IT'S NOT CODIFIED
VERY WELL.
LIKE MOST PEOPLE I KNOW DON'T
QUITE KNOW WHAT THEY NEED TO
WRITE IN THESE STATEMENTS.
OTHER THAN OBVIOUSLY WE MAKE
EVERY EFFORT TO BE RIGOROUS.
SOMETIMES, YOU KNOW IF IT'S A
STRAIGHT STATISTICAL ANALYSIS,
IT'S EASIER TO CODE GUY WHAT YOU
HAVE TO DO.
SAMPLE SIZE, SOMETHING YOU LIKE
FOR EFFECT.
BUT IT ISN'T ALWAYS CLEAR IN
BASIC SCIENCE WHAT CONSTITUTES
RIGOR.
SO I'D JUST SAY, I KNOW ON STUDY
SECTION, THIS WAS A VERY
FRUSTRATING THING.
THERE'S NO UNIFORM STANDARD FOR
SAYING, OH, THIS DOESN'T MEET
THE CRY TIER YO FOR BEING
RIGOROUS OTHER THAN I'VE GOT A
FEELING THIS LOOKS KIND OF
SLOPPY.
BUT IT'S ONE THING TO MAKE A
STATEMENT THAT I'M GOING TO TRY
AND NOT BE SLOPPY BUT I DON'T
KNOW HOW THE NIH EVEN EXPECTS US
HOW TO TREAT THIS, REALLY, AS
THE REVIEWER-BASED SIDE.
>>I WILL SAY THAT WE TRY TO
PROVIDE GUIDANCE, WE HAVE
GUIDANCE ON OUR WEBSITE ON WHAT
TO INCLUDE IN GRANT
APPLICATIONS.
WE'RE ALSO DEVELOPING THIS
EDUCATIONAL RESOURCE THAT WE'RE
HOPING WILL BE USED WIDELY ONCE
IT IS FULLY DEVELOPED.
IT IS CURRENTLY JUST, YOU KNOW,
UNDER DEVELOPMENT AND SO WE
DON'T HAVE ANYTHING YET TO SHARE
ABOUT THAT.
BUT IN ORDER TO PROVIDE PEOPLE
THIS GUIDANCE SO THEY CAN'T SAY
I JUST DON'T KNOW WHAT I'M
SUPPOSED TO BE DOING.
SO WE'RE TRYING TO ATTACK THIS
FROM MULTIPLE ANGLES.
>>I'M VERY HAPPY TO HEAR THAT,
DEVON.
THANKS.
ONE OF THE THINGS I'VE ALWAYS
NOTICED IS LIKE JUST IN MY OWN
LAB'S WORK, WHAT TURNS OUT NOT
TO BE REPRODUCIBLE IS LARGELY
BECAUSE OF WHAT I WOULD CALL
HIDDEN VARIABLES.
LIKE WE DIDN'T KNOW THIS WAS AN
IMPORTANT VARIABLE IN AN
EXPERIMENT.
WE WEREN'T PAYING THAT CLOSE
ATTENTION TO THAT ONE, RIGHT?
THAT USED TO BE GENDER IN
ANIMALS, FOR EXAMPLE, NOW THAT'S
PAID ATTENTION TO.
BUT THERE'S A THOUSAND
VARIABLES.
THESE ARE COMPLEX EXPERIMENTS.
AND WHAT I THINK IS VALUABLE IS
YOU NEED TO BE ACCOUNTABLE FOR
EVERY VARIABLE.
SO LIKE IN THOSE STUDIES
PUBLISHED IN 2014 ON A.L.S.
TRIALS, A LOT OF THAT COULD HAVE
BEEN JUST LITERALLY THEY DIDN'T
KNOW THAT THAT WAS THE CONDITION
THEY USED.
NOW THAT WASN'T THE SAME
CONDITION THAT THE 2014 PAPER
DID, IT DOESN'T MEAN THEY
ACTUALLY WERE RIGHT.
IT'S JUST THEY USE ADD DIFFERENT
CONDITION.
SO THE REPRODUCIBILITY COULD
HAVE COME FROM A NUMBER OF
THINGS.
IT COULD BE STRAIGHT SLOPPINESS,
COULD HAVE BEEN CHEATING, ALL
THOSE THINGS, BUT I KNOW JUST
WITH MY OWN LAB, IT'S NOT THOSE
THINGS, IT'S JUST -- SOMEONE
DOES IT A DIFFERENT WAY ONE DAY,
THEY GET A DIFFERENT RESULT.
WHY IS THAT DIFFERENT?
SO WE MIGHT DIG INTO IT.
BUT I THINK THAT'S ACROSS THE
BOARD, AND SO JUST NEEDING TO BE
ACCOUNTABLE FOR AS MANY DETAILS
OF THE METHODS YOU USE, I WOULD
SAY IS THE MOST IMPORTANT THING
WE CAN DO.
>>MM-HMM.
I WOULD JUST LIKE TO SAY BRIEFLY
THAT I ABSOLUTELY AGREE WITH
THAT.
THAT'S ONE REASON THAT WE REALLY
PUSH FOR RIGOR AND TRANSPARENCY
REPORTING WHAT YOU DID SO OTHER
PEOPLE CAN INTERPRET IT, INSTEAD
OF REPRODUCIBILITY, PER SE,
BECAUSE THERE ARE MULTIPLE
REASONS, THEY AREN'T ALWAYS YOU
DID SOMETHING WRONG, THAT
SOMETHING DIDN'T REPRODUCE.
SO WE DEEMPHASIZE THAT.
>>SHI?
>>TIM, I JUST WANT TO -- OF
COURSE I -- EVERYTHING YOU SAY
IS EXACTLY RIGHT.
AND THE QUESTION IS, OF COURSE,
WHAT DID YOU DO IN REVIEW?
ONE OF THE THINGS THAT WE PUSHED
FOR AND WE WERE PARTIALLY
SUCCESSFUL IS THE FOCUS ON THE
KEY SUPPORTING DATA.
DURING REVIEW.
BECAUSE IF THAT COMES FROM THE
LAB, PREVIOUS WORK OF THE LAB,
THAT'S WHERE YOU COULD REALLY
EVALUATE IF WORK IS RIGOROUS OR
NOT.
WHAT IS BEING PROMISED TO BE
DONE, IT CAN BE PROMISED, RIGHT?
AND AS FAR AS, YOU KNOW,
CONFOUNDING VARIABLES THAT WE
DON'T CONTROL, OBVIOUSLY
THAT'S -- YOU KNOW, THAT'S THE
BREAD AND BUTTER OF SCIENCE, BUT
THERE ARE MEASURES WE CAN DO TO
MINIMIZE THAT, RIGHT, AND THAT
IS BY RANDOMIZING, BY INCLUDING
SUFFICIENT LARGE SAMPLE SIZES SO
IF THE VARIABILITY IS JUST A
RANDOM ONE, IT WILL BE UNDER
CONTROL.
>>THANK YOU.
ONE OF THE THINGS THAT I KNOW
SOME JOURNALS HAVE ADOPTED,
WHICH WAS A POSITIVE THING, IS
JUST THEY INSIST THE METHODS BE
MUCH MORE EXTENSIVE, THEY'LL
SAY, LISTEN, YOU HAVE TO SAY
WHERE YOU GOT EVERY REAGENT
BECAUSE THAT'S ANOTHER VARIABLE
THAT COULD CHANGE EVERYTHING.
YOU KNOW, YOU HAVE TO SAY THE
TEMPERATURE OF THE EXPERIMENT
THAT YOU DID.
THAT'S OFTEN NOT REPORTED.
SO I'M HAPPY TO LISTEN TO YOU,
SHAI, I CONSIDER YOU THE
GODFATHER OF RIGOR, SO --
>>I'LL HOLD MYSELF BACK THERE.
LOUISE?
>>I JUST WANT TO MAKE A
STATEMENT, ESPECIALLY ABOUT,
LIKE, YOU KNOW, THE SEX GENDER
ISSUE.
AND IT'S TRUE, A LOT OF TIMES WE
DON'T KNOW WHAT THE VARIABLE IS,
WE DON'T PAY ATTENTION TO IT,
AND THEN WHEN YOU ACTUALLY, YOU
KNOW, EXAMINE IT, YOU SEE HUGE
DIFFERENCES.
I THINK REPORTING IS ONE THING.
BUT THE STRUCTURE OF THE SPAN,
FOR EXAMPLE, AND MAYBE WALTER OR
SOMEBODY CAN TALK ABOUT THAT,
THEY JUST COMPLETED SPAN ONE,
WHICH WAS THE SAME SIMILAR
PROBLEM TO THE A.L.S., IS WHEN
WE TRY STROKE NEUROPROTECTANTS,
THAT ONE LAB SAID IT'S
PROTECTIVE, OTHER LABS WON'T
FIND IT'S PROTECTIVE.
SO NIH SET UP A WAY TO DO A VERY
LARGE, 2500-MICE STUDY IN SIX
SITES THAT WAS COMPLETELY
BLINDED, AND THAT IS PROBABLY
THE BEST WAY TO GO.
I MEAN, WE HAVE OTHER PROBLEMS
WITH TRANSLATING IN STROKE,
WHETHER THEY WORK AT ALL IN
HUMANS, BUT ONE COMPOUND CAME
OUT AT ALL SIX SITES AS
POSITIVE, AND THE OTHERS DID
NOT.
SO ALTHOUGH REPRODUCIBILITY MAY
BE A BIT BORING, IF YOU'RE
REALLY GOING TO MOVE THINGS NA A
CLININTOA CLINICAL TRIAL, THEY'O
BE REPRODUCED.
BECAUSE IF YOU CAN'T FIND THE
SAME FINDING IN BALTIMORE THAT
YOU CAN IN, YOU KNOW, TENNESSEE,
THEN IT PROBABLY IS NOT A GREAT
THING TO INVEST A LOT OF MONEY
IN.
AND I SOMETIMES SEE EVEN THE
CLINICAL TRIALS IN STROKE, WE DO
STILL DO THAT.
WE DON'T HAVE THIS ROBUST, YOU
KNOW, RIGOROUS PRE-CLINICAL TO
CLINICAL TRANSLATION, AND I
THINK YOU CAN TELL PEOPLE TO DO
POWER ANALYSIS AND SAY THEY'RE
RANDOMIZING AND SAY THEY'RE
DOING ALL THESE THINGS, BUT ARE
THEY ACTUALLY DOING IT?
BUT REPRODUCIBILITY IS COSTLY,
BUT IT'S A LOT LESS COSTLY THAN
SPENDING $20 MILLION ON A
CLINICAL TRIAL THAT IS THE NO
GOING TO WORK.
>>I THINK I'M GOING TO CALL IT
THERE, BUT DO SEND US COMMENTS
ABOUT THIS IF YOU HAVE THEM.
YOU CAN SEND THEM TO ME, I'LL
FORWARD THEM TO SHAI, DEVON,
WALTER. BUT I THINK ALL THESE
COMMENTS HAVE BEEN GREAT.
WHY DON'T WE HAVE A MOTION,
AGAIN, THIS IS STILL IN THE
CONCEPT STAGE, IT'S STILL BEING
DEVELOPED, AND YOUR COMMENTS
HAVE BEEN REALLY THOUGHTFUL, I
THINK.
WE NEED A MOTION FOR THIS
CONCEPT TO GO FORWARD OR NOT.
>>SO MOVED.
>>OKAY.
I ASSUME THAT'S GO FORWARD.
THERE'S A SECOND FROM GINA, AND
YOU CAN VOTE IN THE CHAT.
NEXT WE HAVE THREE MORE
PRESENTATIONS.
FIRST VICKIE WIDIMORE, PROGRAM
DIRECTOR IN OUR CHANNELS,
SYNAPSE AND CIRCUITS, I FORGET
THE FULL NAME, CLUSTER IS GOING
TO DISCUSS A REISSUE OF THE
MECFS CHRONIC FATIGUE SYNDROME
COLLABORATIVE RESEARCH CENTERS.
THIS IS A REISSUE.
WE THOUGHT IT WOULD BE WORTH AT
LEAST PRESENTING TO YOU SO
YOU'RE AWARE.
IS VICKIE THERE?
THERE SHE IS.
>>YES, CAN I HAVE MY SLIDE,
PLEASE?
THANK YOU.
SO YES, WE'RE PRESENTING THE
CONCEPT TO REISSUE
RFA-NS-22-019, WHICH IS A RFA, A
FUNDING ANNOUNCEMENT TO SUPPORT
COLLABORATIVE RESEARCH CENTERS
WITH RESEARCH FOCUSED ON MYALGIC
ENCEPHALOMYELITIS CHRONIC
FATIGUE SYNDROME OR ME/CFS.
THE OVER ARCHING GOAL IS TO
SUPPORT A NETWORK OF CENTERS
THAT WORK COLLABORATIVELY TO
DEFINE THE CAUSES OF, DISCOVER
AND IMPROVE TREEFMENTS OF MECFS
AND A MORE IMMEDIATE GOAL IS TO
RAPIDLY ADVANCE SYNERGISTIC
INTERDISCIPLINARY RESEARCH
PROGRAMS AND WHILE SERVING AS
LOCAL RESOURCES AND NATIONAL
LEADERS IN ME/CFS RESEARCH.
SO AS THOSE OF WHO HAVE BEEN ON
COUNCIL MAY KNOW, WE FUNDED
THREE CENTERS THAT ARE CURRENTLY
IN NO COST EXTENSION AND FUNDED
A DATA MANAGEMENT COORDINATING
CENTER THAT HAS PUT A VERY
ROBUST DATA SHARING PORTAL IN
PLACE FOR CHRONIC FATIGUE
SYNDROME RESEARCH DATA AND SO
WHAT WE'RE LOOKING TO DO IS
SOLICIT APPLICATIONS TO SUPPORT
THREE CENTERS.
THE DATA MANAGEMENT COORDINATING
CENTER IS SUPPORTED OFF OF A
DIFFERENT RFA.
JUST A COUPLE OF THINGS TO NOTE
IS THAT THE CURRENTLY FUNDED
CENTERS HAVE REALLY MADE VERY
SIGNIFICANT PROGRESS IN
UNDERSTANDING THE UNDERLYING
PATHOPHYSIOLOGY OF ME/CFS BUT
MORE RESEARCH IS NEEDED TO
REALLY MOVE THIS RESEARCH
FORWARD SUCH THAT IT CAN MOVE
INTO CLINICAL TRIALS, AND AS WAS
ALLOWED IN THE PREVIOUS RFA
22-019, WE WOULD ALSO ALLOW THE
CENTERS TO INCLUDE A COHORT OF
INDIVIDUALS WITH LONG COVID WITH
POST-ACUTE SEQUELAE OF
SARS-COV-2 OR LONG COVID AS LONG
AS RESEARCH IS FOCUSED ON
OVERLAPPING SYNDROMES BETWEEN
INDIVIDUALSINDIVIDUALS WITH LOND
ME/CFS.
>>I WANT TO MAKE SURE WE SAVE
THE COMMENTS IN THE CHAT AFTER
THE MEETING.
YOU DON'T HAVE TO ANSWER.
OKAY.
QUESTIONS ABOUT THIS RENEWAL?
DO YOU HAVE YOUR HAND UP,
LOUISE?
NO?
>>SORRY.
IT WAS STILL HUNG UP.
>>OKAY.
THIS WAS A LITTLE BIT MORE OF AN
FYI, JUST TO LET YOU KNOW THAT
THESE CENTERS ARE UP.
WE WANT MORE OF THEM.
THEY HAVE HAD SOME
ACCOMPLISHMENTS, ET CETERA.
ANY OTHER QUESTIONS?
OKAY.
I'M GOING TO TRY TO DO A SINGLE
VOTE AT THE END AS OPPOSED TO
VOTING AT EVERY SINGLE ONE OF
THESE.
OKAY.
NEXT, WE HAVE AN INITIATIVE ON
ANOTHER SOMEWHAT COVID-RELATED
INITIATIVE THAT LUMI ADAMS IS
GOING TO PRESENT.
ARE YOU THERE?
>>YES, THANK YOU.
MAY I HAVE THE SLIDES?
THANK YOU SO MUCH.
SO THE DIVISION OF CLINICAL
RESEARCH IS PROPOSING A NEW
CONCEPT ENTITLED UNDERSTANDING
NEUROLOGICAL EFFECTS OF COVID-19
AND POST-ACUTE SEQUELAE OF
SARS-COV-2 INFECTION, OR PASC.
THE COLEADS OF THIS CONCEPTS ARE
DR. BARBARA KARP AND MYSELF.
SO AS PROBABLY MOST OF US KNOW,
NEUROLOGICAL CONDITIONS ARE
EXTREMELY COMMON IN PEOPLE
DIAGNOSED WITH COVID-19.
FOR EXAMPLE, IT HAS BEEN
REPORTED THAT MORE THAN 80% OF
HOSPITALIZED PATIENTS WILL HAVE
AT LEAST ONE NEUROLOGICAL
SYMPTOM, LIKE HEADACHES,
ENCEPHALOPATHY, SEIZURES,
GUILLAIN-BARRE SYNDROME AND
STROKE ARE SOME OF THE COMMON
NEUROLOGICAL MANIFESTATIONS
DURING THE ACUTE PHASE.
BUT NOT ONLY THAT, MORE THAN
HALF OF THE PEOPLE DIAGNOSED
WITH COVID-19 CONTINUE TO HAVE
PERSISTENT SYMPTOMS SUCH AS
BRAIN FOG, NEUROPATHIC PAIN,
MYALGIA, FATIGUE, DISOUGHT NOME
YA, MOOD DISORDERS, BEYOND ACUTE
STATES, AND THAT IS A CONDITION
THAT'S KNOWN AS PASC OR CLOAK OG
COVID.
THERE IS GROWING EVIDENCE THAT
COVID-19 MAY INCREASE THE RISK
OF DEVELOPING NEUROLOGICAL
CONDITIONS LIKE ALZHEIMER'S
DISEASE, MOVEMENT DISORDERS AND
EPILEPSY IN THE LONG TERM.
SO WE ALSO KNOW THERE ARE
HUNDREDS IF NOT THOUSANDS OF
ONGOING COVID-RELATED STUDIES
AROUND THE WORLD, INCLUDING THE
NIH RECOVER INITIATIVE TO BETTER
CHARACTERIZE COVID AND PASC
SYMPTOMS, BUT ALSO TO UNCOVER
PATHOPHYSIOLOGY INCLUDING LARGER
SCALE SURVEYS, REVIEW OF LARGE
ELECTRONIC HEALTH RECORDS,
PROSPECTIVE COHORT STUDIES
INCLUDING COLLECTION OF
BIOSPECIMENS.
HOWEVER, MECHANISMS UNDERLYING
NEUROLOGICAL MANIFESTATIONS
CONTINUES TO BE VERY POORLY
UNDERSTOOD, AND IT IS A
BOTTLENECK FOR THE DEVELOPMENT
OF EFFECTIVE TREATMENTS AND
PREVENTION.
OBVIOUSLY COVID RELATED
MECHANISTIC APPLICATIONS WILL
CONTINUE TO BE SUBMITTED VIA
PARENT ANNOUNCEMENTS, HOWEVER,
MANY OF THESE APPLICATIONS ARE
NOT RELEVANT TO THE MISSION OF
NINDS, AND THAT LEADS TO A BIG
GAP IN RESEARCH FOCUSED ON
NEUROLOGICAL ASPECTS OF THE
PANDEMIC.
SO HERE WE ARE PROPOSING TWO NEW
RFAs UNDER THIS SAME CONCEPT
TO BETTER UNDERSTAND
NEUROPATHOGENESIS OF COVID AND
PASC.
MORE SPECIFICALLY, THESE RFAs
WOULD BE INVITING APPLICATION
ADDRESSED IN THE MECHANISM
BIOMARKERS AND PA THROW
PHYSIOLOGY OF NEUROLOGICAL
MANIFESTATIONS ASSOCIATED WITH
COVID AND PASC.
APPLICATIONS PROPOSING
SCIENTIFICALLY COMPELLING
PATHWAYS TO PREVENT THE
DEVELOPMENT OF PASC OR TO
ACCELERATE DEVELOPMENT OF
EFFECTIVE TREATMENTS FOR
PASC-RELATED NEUROLOGICAL
COMPLICATIONS ARE OF PARTICULAR
INTEREST FOR OUR NEW CONCEPT.
HOWEVER, INTERVENTIONAL TRIAL
TESTING SAFETY AND EFFICACY WILL
NOT BE ALLOWED AT THIS TIME.
WE ARE PROPOSING R01 AND R21 AS
MECHANISMS FOR FUNDING WITH TWO
SEPARATE RECEIPT DATES.
AND WE WOULD LIKE TO REQUEST A
SPECIAL EMPHASIS PANEL TO BRING
THE APPROPRIATE EXPERTISE TO THE
REVIEW.
WE REALLY BELIEVE THAT NIH
HAS -- NINDS HAS A HUGE
POTENTIAL TO MAKE AN IMPACTFUL
PROGRESS IN THE AREA AND WITH
THE PURPOSE OF THIS TO OUR
RFAs.
WITH THAT, I WOULD LOVE TO TAKE
QUESTIONS OR COMMENTS.
>>THANKS, LUMY.
GOOD PRESENTATION.
ANY QUESTIONS FOR HER?
>>I JUST WANT TO MOVE THAT
WE --
>>OKAY.
SAVE THE MOTION FOR A LITTLE.
I JUST HATE DOING A MOTION ON
EVERY SINGLE THING.
OKAY.
AGAIN, ANY OTHER QUESTIONS ABOUT
THAT?
OKAY.
THANKS.
AND THE LAST CONCEPT IS FROM
STEVE KORN, THE HEAD OF OUR
TRAINING OFFICE WHO YOU'VE HEARD
FROM BEFORE.
I SEE HIM STANDING UP THERE.
>>I WANT TO APOLOGIZE, I'VE GOT
TWO CAMERAS IN MY OFFICE AND I
CAN'T CONTROL WHICH ONE IS
LOOKING AT ME.
>>IT'S GOOD TO SEE YOU FROM A
DIFFERENT PERSPECTIVE.
>>YOU GET A PROFILE.
SO THANKS, THIS IS THE END FOR
TODAY.
AND I WANT TO TALK TO YOU ABOUT
A NEW INITIATIVE FOR WORKFORCE
DEVELOPMENT, AND THIS IS TO
DEVELOP AN EMERGENCY MEDICINE
CLINICCLINICIAN-SCIENTIST WORKF.
AND IF WE CAN GO TO THE NEXT
SLIDE, THERE'S A TERRIFIC NEED
FOR EMERGENCY MEDICINE
RESEARCHERS.
THEY'RE ON THE FRONT LINE OF
LOTS OF CONDITIONS THAT ENTER
THE HOSPITAL.
I HAVE SOME NUMBERS HERE, YOU
CAN ALL LOOK ON LOOK AT THE NUI
WON'T READ THEM TO YOU, BUT A
LARGE NUMBER OF PEOPLE GOING
INTO THE EMERGENCY ROOM GO IN
WITH NEUROLOGICAL COMPLAINTS OR
SERIOUS CONDITIONS, AND I'LL
JUST POINT OUT ONE NUMBER TO
YOU, WHICH IS A LITTLE BIT
SKEWED GIVEN THAT IT'S 2022, BUT
NINDS MADE EIGHT GRANT AWARDS TO
EMERGENCY MEDICINE CLINICIANS IN
A YEAR.
SO WE'RE REALLY IN NEED OF MORE
EMERGENCY MEDICINE CLINICIANS TO
DO RESEARCH.
MOST OF THE RESEARCH THEY END UP
DOING IS CLINICAL RESEARCH,
DEALING DIRECTLY WITH THE
PATIENTS.
YOU CAN SEE THE NUMBERS BELOW
JUST FOR CONTEXT, HOW MANY MORE
NEWERNEUROLOGISTS ARE GETTING GS
THAN EMERGENCY MEDICINE
CLINICIANS, JUST TO GIVE YOU
SCOPE.
NEXT SLIDE.
SO THE WAY WE LIKE TO DO THIS IS
WITH A NATIONAL PROGRAM THAT'S
MODELED AFTER TWO OTHER
SUCCESSFUL PROGRAMS FOR
WORKFORCE DEVELOP: ONE IS FOR
NEUROSURGEONS AND ONE IS FOR
PEDIATRIC NEUROLOGISTS.
AND YOU ACTUALLY HEARD EARLIER
TODAY THAT HEAL IS USING THE
SAME MODEL TO DEVELOP THEIR
WORKFORCE.
AND TO GIVE YOU AP IDEA -- THESE
PROGRAMS ARE VERY SUCCESSFUL,
AND WHAT REALLY LEADS TO THE
SUCCESS IS THAT THEY'RE NATIONAL
PROGRAMS THAT DRAW FROM THE
ENTIRE COUNTRY, THAT DRAW FROM
THE BEST PEOPLE IN THE ENTIRE
COUNTRY, AND THEY INVOLVE THE
COMMUNITIES, NOT JUST A
DEPARTMENT, AND AND A FEW SUPPG
SCHOLARS.
SO AS YOU CAN SEE THERE ARE
THREE LEVELS OF SUCCESS FOR
THESE PROGRAMS.
THE FIRST IS THE SCHOLARS THAT
ARE BEING SUPPORTED, IN BOTH OF
THESE PROGRAMS ARE OVER 80% HAVE
RECEIVED MAJOR FUNDING AFTER
GOING THROUGH THESE PROGRAMS.
THE SECOND LEVEL OF SUCCESS IS
THAT EVEN PEOPLE WHO APPLY TO
THE PROGRAMS AND ARE NOT FUNDED
ARE DOING VERY WELL, BECAUSE THE
PROGRAMS PROVIDE AN ENVIRONMENT,
A COMMUNITY, A MENTORING NETWORK
FOR EVERYBODY, NOT JUST FOR
THOSE THAT ARE FUNDED.
AND THEN THE THIRD LEVEL WHICH
REALLY ONE PROGRAM THAT'S OLD
ENOUGH TO TAKE A LOOK AT IT, BUT
IN NEUROSURGERY, THERE'S WHAT WE
CALL -- THERE'S WHAT WE'VE
CALLED THE KNOCK-ON EFFECT,
WHERE IT'S HAD A FAR GREATER
REACH THAN JUST THE PEOPLE THAT
HAVE BEEN INVOLVED IN THE
PROGRAM, AND THAT'S BECAUSE SOME
OF THE PEOPLE RUNNING THE
PROGRAM, THE FACULTY, ARE --
THERE'S A HUGE NUMBER OF
FACULTY, 60, 70, 80 PEOPLE
INVOLVED FROM THE COMMUNITY THAT
ARE INVOLVED IN RUNNING THESE
PROGRAMS.
AND WHAT IT DOES IS IT ENERGIZES
EVERYBODY TO GET INVOLVED IN
DOING MORE RESEARCH.
SO WE CAN GO TO THE NEXT SLIDE.
SO SOME OF THE FUNDAMENTAL
FEATURES, THERE'S JUST ONE
PROGRAM RUN BY ONE OR MORE
PIs.
THERE'S A NATIONAL ADVISORY
COMMITTEE WHICH IS REALLY THE
GROUP THAT RUNS THE PROGRAM,
IT'S NOT RUN BY A PI, IT'S RUN
BY A GROUP OF PEOPLE.
AND THIS COMMITTEE IS
DIVERSIFIED IN ALL DIFFERENT
KINDS OF WAYS.
YOU CAN SEE SOME OF THE WAYS
I'VE PUT UP ON THE SCREEN.
BUT THE IDEA IS TO MAKE THIS
OPEN TO EVERYBODY AND TO
ELIMINATE BIAS THAT MIGHT BE
BASED ON INSTITUTION OR TRAINING
OR THE RESEARCH SUBJECT.
YOU SELECT FROM THE BEST CANNED
TAITCANDIDATES IN THE COUNTRY, D
IT'S COMPETITIVE, THEY'RE NOT
APPLYING TO NIH, THEY'RE
APPLYING TO A PROGRAM WHERE
COMMUNITY ARE THE ONES SELECTING
THE BEST PEOPLE.
THE PROGRAM PROVIDES SITE VISITS
AND WEBINARS AND THEY HAVE
STRUCTURED EFFORTS TO ENHANCE
DIVERSITY WITHIN THE SPECIALTY.
THERE'S AN ANNUAL MEETING FOR
EACH OF THESE GROUPS THAT
PROMOTES COMMUNITY BUILDING,
WHICH IS A HUGE PART OF THESE
PROGRAMS.
IT JUST GETS THE ENTIRE
COMMUNITY INVOLVED.
AND REALLY WHAT IT DOES IS IT
MAKES THE PIs AND PROGRAM
ACCOUNTABLE TO THE COMMUNITY,
NOT TO THEMSELVES, REALLY.
AND AGAIN, THE FIRST GOAL IS TO
GET THE SCHOLARS TO FUNDING, BUT
A SECONDARY GOAL IS TO GET
EVERYBODY INVOLVED IN FUNDING.
SO SPECIFIC TO THIS PROGRAM, WE
ARE GOING TO RUN IN, NIDA AND
NIA HAVE SIGNED ON AND MAKE
TRIBUTE.
THIS WOULD BE A STANDARD
INSTITUTIONAL PROGRAM THAT'S
MADE FOR THESE EMERGENCY
MEDICINE CLINICIANS IN THEIR
CIRCUMSTANCES, SO THEY'D GET
THREE YEARS OF SUPPORT, 75%
REQUIRED EFFORT, AND WE WOULD
REQUIRE ANYBODY FUNDED BY THIS
AWARD IS GIVEN AT LEAST 50%
PROTECTED TIME FOR RESEARCH FOR
A SUBSEQUENT TWO-YEAR PERIOD TO
GET THEM TIME TO GET FUNDING IF
THEY HAVEN'T ALREADY.
THAT'S A REQUIREMENT THAT THE
CHAIR HAS TO PROMISE WITH
INSTITUTION SIGN-ON.
A LITTLE BIT OF SPECIFICITY,
BECAUSE OF THE KINDS OF RESEARCH
EMERGENCY MEDICINE CLINICIANS
ARE LIKELY TO DO, WHICH IS
HEAVILY WEIGHTED TOWARDS
CLINICAL RESEARCH AND CLINICAL
TRIALS, WE WOULD REQUIRE
CLINICAL TRIAL EXPERTISE, AND I
FORGOT TO PUT ON THIS SLIDE BUT
WOULD ALSO REQUIRE EXPERTISE IN
ETHICS ON THIS COMMITTEE.
AND THE MEASURES ARE WHAT I
TALKED ABOUT BEFORE, THE SUCCESS
IS MEASURED BY, NUMBER ONE, THE
SCHOLARS GETTING FUNDED, NUMBER
TWO, EVERYBODY ELSE INVOFERLED
GETTING FUNDED, AND NUMBER
THREE, THE ENTIRE COMMUNITY
INCREASING THEIR FUNDING.
AND THE GOAL OF A PROGRAM LIKE
THIS IS TO EVENTUALLY CREATE A
SELF-SUSTAINING ENTITY WHERE
THEY DON'T NEED SPECIAL PROGRAMS
ANYMORE, THEY'RE JUST UP AND
RUNNING.
SO I'LL STOP WITH THAT, TAKE ANY
QUESTIONS IF THERE ARE ANY.
>>OKAY.
COULD WE TAKE DOWN THE SLIDE.
ANY QUESTIONS FOR STEVE?
TIM?
>>THANK YOU.
I'M NOT A PHYSICIAN BUT I'M
SURROUNDED BY THEM.
THERE'S DOZENS OF CLINICAL
DEPARTMENTS AROUND ME AND I'VE
ALWAYS WONDERED WHAT MOTIVATES
THE CLINICAL CHAIR TO HAVE
PHYSICIANS THAT COULD BE EARNING
CLINICAL DOLLARS DOING RESEARCH,
WHERE THEY CLEARLY ARE BRINGING
ESSENTIALLY THEY'RE COSTING THE
PLACE MONEY BECAUSE RESEARCH
COSTS MONEY, IT DOESN'T MAKE
MONEY FOR THEM.
AND MY CONCLUSION HAS ALWAYS
BEEN, IT'S SORT OF BRAGGING
RIGHTS, LIKE THEY LIKE HAVING
SCHOLARSHIP, IT RAISES THEIR
RANKINGS, THEY'RE MORE
ATTRACTIVE, AND YOU KNOW,
CLEARLY THAT -- AND I'VE NEVER
SEEN ANY RESEARCH IN AN
EMERGENCY MEDICINE DEPARTMENT.
CERTAINLY IT'S NOT LOCAL HERE
WHERE I AM IN NEW YORK CITY, I
DON'T SEE THAT.
SO IT SEEMS LIKE THERE HAS TO BE
A CULTURAL SHIFT, LIKE YOU HAVE
TO MAKE IT SUCH THAT THE CHAIRS
WILL FEEL LIKE THEY HAVE
BRAGGING RIGHTS WHEN THEY HAVE
THESE THINGS THERE.
AND I THINK ONCE THAT'S THERE,
THEN THEY'LL OPEN THE DOORS UP
AND ALLOW THESE PEOPLE TO DO IT.
BUT UNTIL THEN, THEY PROBABLY
WON'T ALLOW THEM TO.
>>SO LET ME -- I'VE GOT TWO
ANECDOTAL WAYS TO TALK ABOUT
THAT, TIM.
NUMBER ONE, THIS ABSOLUTELY
RELIES ON PRIDE.
BECAUSE AT BEST, IF THE
COMMUNITY ISN'T INTERESTED, AT
BEST WE'RE GOING TO HAVE 15 MORE
PEOPLE DOING RESEARCH.
THE GOAL IS NOT TO JUST HAVE 15
MORE PEOPLE DOING RESEARCH, THE
GOAL IS TO GET THE WHOLE
COMMUNITY TO EMBRACE THE NEED
FOR RESEARCH IN THEIR SPECIALTY.
AND WHAT DOES THAT IS WHAT YOU
SAID.
YOU KNOW, YOU CALL IT BRAGGING
RIGHTS, BUT THEY WANT TO BE A
PLAYER IN THIS NEW ENDEAVOR OF
DOING RESEARCH TO SOLVE
PROBLEMS.
THE OTHER THING I CAN SAY TO
THAT IS WHEN I WAS DOING MY OWN
LITTLE BIT OF RESEARCH AND
LOOKING AT WEBSITES, EVERY
EMERGENCY ME SIN DEPARTMENT THAT
I LOOKED AT JUST ON THE
INTERNET, THEY LED WITH, "WE'RE
A GREAT RESEARCH DEPARTMENT.
COME DO EMERGENCY MEDICINE
HERE."
AND, YOU KNOW, THAT KIND OF MADE
ME THINK, WELL, WHY DO WE NEED
THIS IF THERE ARE ALWAYS SO MANY
GREAT DEPARTMENTS OUT THERE?
BUT EMERGENCY MEDICINE SEEMS TO
WANT THIS.
AND, YOU KNOW, WE'LL SEE WHAT
HAPPENS.
>>I'M GLAD TO HEAR IT.
THANK YOU, STEVE.
>>OKAY.
SAMEER?
>>THANKS, STEVE.
LOVE THAT PRESENTATION.
REAL QUICK, I JUST WILL REPLY TO
TIM, THERE'S TWO ACTUAL
NEUROLOGY CHAIRS HERE, SO WE'LL
SEE WHAT THEY THINK.
I AM NOT A CHAIR, BUT TO ANSWER
YOUR QUESTION, SOME OF IT, YEAH,
IS BRAGGING RIGHTS, BUT
HONESTLY, DEPARTMENT CHAIRS ARE
THEMSELVES ACADEMICIANS, SO, YOU
KNOW, THE PURSUIT OF SCIENCE AND
RIGOR OF SCIENCE AND EXCELLING
AND HAVING THE DEPARTMENT EXCEL
IN WHATEVER FIELD IS SOMETHING
THAT IS INNATE TO THEM.
AND IF THEY DIDN'T CARE ABOUT
THAT, THEY'D BE IN A PRIVATE
PRACTICE OR DOING SOMETHING
DIFFERENTLY.
I MEAN, I'VE BEEN LUCKY ENOUGH
TO BE HIRED BY CHAIRS WHO WANT
ME TO DO SCIENCE AND SPEND HALF
MY TIME NOT MAKING MONEY FOR THE
HOSPITAL BUT DOING, YOU KNOW,
DOING SCIENCE.
ANYWAY, BUT THE POINT I WAS
GOING TO MAKE IS A QUICK ONE TO
STEVE JUST THAT AS A PRODUCT OF
A K12 PROGRAM THAT STEVE STARTED
MANY YEARS AGO, I DON'T KNOW,
MORE THAN A -- I DON'T KNOW,
MORE THAN 15, 17, 18 YEARS AGO
FOR NEUROSURGERY, I'LL JUST SAY
THAT, YOU KNOW, IT WAS A
FANTASTIC EXPERIENCE FOR ME BACK
THEN, AND I THINK IT REALLY DOES
INTRODUCE THESE YOUNG PEOPLE TO
WHAT THE NIH MACHINE IS LIKE,
WHAT THE PROCESS IS LIKE, AND
HAVING -- I TALK AT THESE
CONFERENCES EVERY YEAR AND I
JUST SAY, HAVING DR. KORN IN
YOUR CORNER REALLY MAKES A
DIFFERENCE AT THAT YOUNG STAGE
IN ONE'S CAREER.
SO I THINK THESE PROGRAMS, FOR
ME ANYWAY AND A LOT OF PEOPLE
THAT I KNOW AS YOUNG
NEUROSURGEONS BACK THEN WAS
EXTREMELY EFFECTIVE, AND I CAN
ONLY ASSUME THE SAME WOULD BE
TRUE FOR SOMETHING LIKE THIS.
>>THANKS, SAMEER.
MAYBE TWO MORE QUESTIONS.
WE HAVE ED AND THEN --
>>JUST TO ADDRESS QUICKLY THE
COMMENT ABOUT WHY WOULD
DEPARTMENTS WANT TO DO THIS,
GIVEN THE DEMAND WE ALL HAVE TO
MAKE MONEY, I THINK ONE OF THE
ISSUES THAT IS COMING UP AT
LEAST AT VANDERBILT WHERE OUR
NEW INTERIM CHAIR IS PRIMARILY A
RESEARCHER IS THAT THE WHOLE
ENTERPRISE, THE WHOLE MEDICAL
SCHOOL SEES THE EMERGENCY ROOM
AS A CONDUIT TO CLINICAL
RESEARCH, AND SO EMERGENCY
MEDICINE IS OFTEN AN ENTRY POINT
FOR DOING ALL SORTS OF DIFFERENT
RESEARCH PROJECTS, AND SO
INCLUDING IN NEUROLOGY.
SO HAVING A REALLY STRONG
RESEARCH GROUNDING IN THE
EMERGENCY MEDICINE DEPARTMENT
HELPS THE WHOLE INSTITUTION WITH
ITS RESEARCH MISSION.
SO I THINK THAT PERHAPS SUPPORT
FROM THE DEANS FOR THE CHAIRS IN
EMERGENCY MEDICINE TO SUPPORT
OUR RESEARCH CAN BE REALLY
HELPFUL IN THIS SITUATION.
BUT I'M VERY EXCITED ABOUT THIS
PROPOSAL.
IT SEEMS LIKE IT'S NEEDED AND I
THINK IT WILL BE REALLY WELCOMED
ACROSS THE COUNTRY.
>>THANKS A LOT, ED.
LAST, JIN-MOO.
>>I'M JUST JUMPING IN TO ADD TO
WHAT EVERYONE ELSE HAS BEEN
SAYING IN RESPONSE TO TIM'S
QUESTION ABOUT WHY WE IN
ACADEMIC MEDICINE SUPPORT
RESEARCH.
ONE THING I'D SAY IS THAT PART
OF THE ACADEMIC MISSION IS TO DO
RESEARCH.
THE RESEARCH BRINGS PRESTIGE TO
THE DEPARTMENT, WHICH ATTRACTS
CLINICIANS HERE.
SO THERE'S A VIRTUOUS CYCLE OF
THE RESEARCH BRINGING THE
CLINICIANS.
THE CLINICIANS BRING IN THE
REVENUE THAT SUPPORTS THE
RESEARCH.
SO IT'S THIS VIRTUAL CYCLE THAT
SUPPORTS ONE ANOTHER.
AND YOU'LL PROBABLY NOTICE THAT
EVERY FEBRUARY, THE CHAIRS RUN
TO THE BLUE RIDGE NIH RANKING,
BECAUSE THAT'S A REALLY
IMPORTANT PART OF HOW A
DEPARTMENT PERFORMS, HOW GOOD IS
THE RESEARCH.
IS IT SUPPORTED BY NIH.
SO I THINK IT'S THE PACKAGE DEAL
THAT SEPARATES ACADEMIC MEDICINE
FROM PRIVATE PRACTICE.
IT'S REALLY THAT RESEARCH.
AND SO I ABSOLUTELY SUPPORT THE
K12.
THIS HAS BEEN A FANTASTIC
PROGRAM, AND THE E.D., I THINK
AS A NEW SPECIALTY, REALLY NEEDS
THAT BOOST TOWARDS ITS RESEARCH
YOUNG FACULTY.
>>THANKS A LOT, JIN-MOO.
THAT'S VERY HELPFUL.
OKAY, I HAVEN'T HEARD ANY
COMMENTS ABOUT ANY OF THESE
THREE INITIATIVES.
REMEMBER, WE'RE TALKING ABOUT
THE CHRONIC FATIGUE SYNDROME
CENTER RENEWAL, THE COVID
SEQUELAE, THE NEUROLOGICAL
PHENOTYPES INITIATIVE, AND THEN
LAST ONE.
SO I'M ASSUMING THERE WILL BE A
MOTION TO APPROVE THE THREE OF
THOSE.
THERE'S GINA.
A SECOND?
YOU CAN VOTE ON ALL THREE OF
THESE CONCEPTS IN THE CHAT.
AGAIN, THESE ARE STILL IN THE
CONCEPT STAGE, SO THEY'LL
CONTINUE TO BE DEVELOPED.
IF WE COULD PUT UP THE LAST
SLIDE, THE LAST THING ON THE
AGENDA, WHICH I'M GOING TO GO
THROUGH VERY QUICKLY.
SO THIS IS THE LIST OF THE
CONCEPTS WE'RE NOT PROPOSING TO
DISCUSS.
IF YOU HAVE QUESTIONS, YOU CAN
ASK THEM.
I'LL JUST VERY QUICKLY GO
THROUGH THEM.
IT'S MOSTLY A LIST OF RENEWALS.
IF YOU LOOK AT THE LEFT-HAND
COLUMN HERE.
THERE'S A SECOND SLIDE YOU'LL
SEE IN A MINUTE.
SO THE FIRST FIVE, LET'S SEE,
ONE, TWO, THREE, FOUR, FIVE, OH,
ACTUALLY THE ORDER IS A LITTLE
BIT DIFFERENT.
OH, NO, THE FIRST FIVE ARE ALL
NICINITIATIVES THAT ARE GOING TE
FUNDED IF WE MOVE FORWARD ON
THEM ESSENTIALLY FROM OUR UR
NINDS BASE BUDGET.
AFTER THAT IT'S MOSTLY
INITIATIVES COMING FROM
DIFFERENT FUNDING STREAMS LIKE
BRAIN, ET CETERA.
I DON'T HAVE A POINTER HERE BUT
IF YOU GO THROUGH THEM QUICKLY,
NUMBER ONE IS A PROGRAM THAT
PROVIDES SEED MONEY FOR GROUPS
OF PIs WHO WANT TO APPLY TO
OUR PARKINSON'S UDAL CENTER
PROGRAM.
WE'VE DONE THIS BEFORE, IT SEEMS
TO HAVE BEEN VERY EFFECTIVE.
NEXT IS A RENEWAL OF THE ACTUAL
UDALL CENTER'S PROGRAM, WHICH IS
CONGRESSIONALLY MANDATED.
THIS IS SOMETHING THAT WE WERE
TOLD THAT WE WILL TO HAVE, AND
THEY'VE GONE ON TO BEING
EXTREMELY SUCCESSFUL.
THIRD IS THE BLUEPRINT ENDURE
PROGRAM, WHICH ESSENTIALLY HOW
TO SUMMARIZE IT IN A SENTENCE,
IT HELPS UNDERGRADUATE STUDENTS
FROM DIVERSE BACKGROUNDS TO --
IT ENCOURAGES THEM AND HELPS
THEM GET INVOLVED IN PH.D.
PROGRAMS IN NEUROSCIENCE.
THAT'S BLUEPRINT ENDURE.
THIS IS ALSO A REISSUE.
THE NEXT TWO, NUMBERS 4 AND 5,
ARE ESSENTIALLY REISSUES OF OUR
STANDARD TRAINING GRANT AND
K01 PROGRAMS.
AND THEN LET'S SEE, WHAT'S NEXT
HERE.
NEXT IS -- OKAY, THE ORDER HAS
CHANGED A LITTLE BIT.
OKAY.
NEXT IS THE COUNTER -- IS A
COUNTERACT INITIATIVE.
THIS IS OUR COUNTERTERRORISM
PROGRAM WHICH IS PAID THROUGH A
SEPARATE FUNDING STREAM.
THE ALS -- THIS IS A RENEWAL OF
THE ALS EXPANDED ACCESS
INITIATIVE THAT WE'VE SEEN
BEFORE IN ITS PREVIOUS
ITERATION.
NEXT IS A BLUEPRINT PROGRAM, AND
BLUEPRINT CONSISTS OF ALL THE
NIH INSTITUTES THAT FUND
NEUROSCIENCE.
WE ARE ONE OF THE MAJOR PLAYERS
IN THIS PROGRAM.
IT'S A LONG-STANDING AND VERY
SUCCESSFUL PROGRAM AND NINDS
CONTRIBUTES PARTLY THE BLUEPRINT
BUDGET.
AND ALL OF THE REMAINING
INITIATIVES INVOLVE EITHER
BRAIN, HEAL, OR ADADRD.
AND EACH OF THEM HAS A DEDICATED
FEDERAL SETASIDE.
SO FOR EXAMPLE, YOU CAN SEE THAT
THERE'S THE BRAIN INITIATIVE
LISTED UNDER NUMBER 9, THERE'S
THREE INITIATIVES AFTER THAT,
THE EXPOSOME ALL FOR HEAL, ACUTE
AND CHRONIC PAIN, AND ONE ON
WEARABLE DEVICES.
IF WE COULD GO TO THE NEXT
SLIDE.
I'M NOT GOING TO READ ALL OF
THESE BUT YOU'VE GOT WRITE-UPS
FOR ALL OF THEM IN THE ECB, AND
YOU CAN SEE THAT THE FIRST TWO
ARE HEAL, AND ALL THE REST OF
THEM ARE ESSENTIALLY AD/ADRD
INITIATIVES.
NOW THE THING THAT I'LL SAY IS
THAT ALL OF THESE INITIATIVES
FOR BRAIN, HEAL AND ADRD ARE
VERY EXTENSIVELY VETTED BEFORE
YOU SEE THEM HERE.
THEY'RE VETTED BY GROUPS OF
STAFF, THEY'RE VETTED IN TWO OF
THESE CASES, IN THE CASE OF HEAL
AND BRAIN, BY MULTI-COUNCIL
WORKING GROUPS WHICH HAVE
DISCUSSED THEM IN DETAIL, AND
MOST IMPORTANTLY, THEY'RE VETTED
BY THE INSTITUTE DIRECTORS OVER
THE INITIATIVES -- OF THE
INSTITUTES THAT PARTICIPATED
BRING HEAL, ADRD.
SO WE WEREN'T PLANNING TO DELVE
INTO ANY OF THESE.
WHY DON'T YOU GO BACK TO THE
FIRST SLIDE AGAIN.
BULL -- BECAUSE THE FIRST FIVE
ARE THE ONES THAT REALLY COME
DIRECTLY OUT OF OUR BUDGET.
BUT IF THERE'S ANY QUICK
QUESTIONS, WE CAN HAVE THE
PROGRAM DIRECTORS WHO ARE LISTED
ON THE LAST COLUMN ANSWER THEM.
ANYTHING PEOPLE WANT TO KNOW?
OKAY.
IN THAT CASE, IF THERE ARE
QUESTIONS LATER ON, EMAIL ME AND
I CAN ANSWER THEM.
IN THAT CASE, I NEED AN EN BLOC
MOTION TO APPROVE ALL THE
INITIATIVES ON THESE TWO SLIDES
TO GO FORWARD.
>>MOTION TO APPROVE EN BLOC.
>>NICE JOB, TIM.
I SEE A SECOND, I THINK FROM
GINA.
THINK SHE HAD HER HAND UP.
YOU CAN TAKE THAT SLIDE DOWN,
AND THEN YOU CAN VOTE IN THE
CHAT.
DID YOU JUST SAY SOMETHING,
WALTER?
THERE'S AN ECHO COMING FROM YOU.
I CAN'T HEAR YOU, WALTER.
>>HE GOT RID OF THE ECHO.
>>CAN YOU HEAR ME?
>>NOW WE CAN HEAR YOU.
>>OH, OKAY.
I WAS GOING TO SAY, THAT
IMPRESSIVE LIST IS A TESTIMONY
TO THE AMOUNT OF WORK THE
PROGRAM DIRECTORS ARE DOING.
YOU KNOW, IT'S ONE THING TO BE
ABLE TO, YOU KNOW, GET YOUR
R01 GRANTEE AND TALK TO THEM,
BUT IT'S LIKE COMPLETELY
DIFFERENT TRYING TO PUT THESE
PROGRAMS TOGETHER, AND AS YOU
SAW, MOST OF THOSE PROGRAMS ARE
BEING FUNDED BY THESE SPECIAL
TARGETED FUNDS.
BUT THAT'S REALLY WHERE NINDS
STAFF CAME TO THE FOREFRONT.
THEY ARE BLAZING NEW RESEARCH
AREAS.
>>THERE'S BEEN A HUGE CHANGE
SINCE I GOT HERE, WHICH IS
ESSENTIALLY THESE THREE HUGE
FEDERAL INITIATIVES.
THERE'S ALSO COUNTERACT AS WELL.
WHICH NOW COMPRISE, YOU KNOW,
CLOSE TO HALF OUR BUDGET.
SO THERE'S BEEN AN INCREDIBLE
RAMP UP-UP BY UP BY OUR STAFF TE
THESE WORK AND THAT'S WHAT GINA
WAS APPLAUDING AND WONDERING
ABOUT AND WORRYING ABOUT.
SO YES, PEOPLE ARE STRESSED TO
THE LIMIT AT THE MOMENT AND
WE'RE TRYING TO DO SOMETHING
ABOUT THAT.
OKAY, LOOK, I THINK --
>> -- WORK HERE.
IT'S A GREAT PLACE.
>>YEAH, THAT'S RIGHT.
OKAY.
THANKS TO EVERYBODY.
GOING ONCE FOR LAST COMMENTS?
TWICE.
WE ARE ADJOURNED, AND I'LL SEE
YOU ALL TOMORROW.
>>THANK YOU.
BYE-BYE.