WELCOME THIS IS THE 224th
MEETING OF THE NATIONAL ADVISORY
NEUROLOGICAL DISORDERS AND
STROKE AND THIS IS OUR OPEN
SESSION.
AND HERE AT THE OPEN SESSION WE
GIVE A REPORT, KIND OF A STATE
OF NANDS AND THEN WE HAVE A
NUMBER OF TOPICS THAT HAVE
ARISEN THAT MAY BE INTERESTING
TO YOU.
SOME ARE ACTUALLY REPORTS FROM
THE WORK BEING GROUPS WORKING AS
PART OF COUNCIL AND SO LOOK
FORWARD TO AN EXCITING MORNING
AND AFTERNOON.
I WANT START BY THANKING THOSE
WHO STAYED ON THE COUNCIL
MEETING WHILE WE AWAIT NEW
COUNCIL MEMBERS TO BE APPROVED.
I WANT TO INTRODUCE THE NEW AD
HOCS WE CALL SUBJECT MATTER
EXPERTS AT THIS TIME.
WE HAVE A PROFESSOR IN THE
DEPARTMENT OF NEUROLOGICAL
SURGERY AT CASE WESTERN RESERVE.
EXPERT IN SURGERY AND HAS DONE
GREAT RESEARCH ON CEREBRAL
VASCULAR BLOOD FLOW AND IT'S
RELATIONSHIP TO STROKE.
IT'S A PLEASURE.
GRAY DAVIS.
A PROFESSOR OF NEUROLOGY IN THE
DEPARTMENT OF BIO CHEMISTRY
UNIVERSITY OF CLINICAL RESEARCH
SAN FRANCISCO.
HIS WORK IS FOCUSSED ON
HOMEOSTATIC REGULATION OF
FUNCTION AND SYNAPSE
STABILIZATION IN DEVELOPMENT AND
DISEASE.
PRIOR TO DIRECTOR OF THE
INSTITUTE FOR FUNDAMENTAL
NEUROSCIENCE AT UCSF AND GREAT
TO HAVE YOU ON.
JOE FRENCH IS THE EXECUTIVE
DIRECTOR AND CO-FOUNDER OF
NEUROTECH NETWORK A NON-PROFIT
FOCUSSED ON THE NEUROTECHNOLOGY
INDUSTRY AND FOUNDING MEMBER AND
PRESIDENT OF THE NORTHERN
AMERICAN SPINAL CORD INJURY
CONSORTIUM AND IS THE CHAIR
PERFORM OF THE RESEARCH PROGRAM
CONGRESSIONALLY DIRECTED
MEDICALLY DIRECTED RESEARCH
PROGRAM.
AND TERRY?
GOOD TO HEAR FROM YOU.
WE GO WAY BACK.
TERRY HAS BEEN THE DIRECTOR AT
THE U.S. DEPARTMENT OF DEFENSE
WHERE RESPONSIBLE FOR STRATEGIC
PLANNING, ANALYSIS AND OVERSIGHT
OF DEFENSE PROGRAMS.
HE'S HAD OVER 40 YEARS OF
EXPERIENCE IN MANY FACETS OF THE
MILITARY HEALTH SYSTEM.
HE'S HAD COLLABORATIONS OVER THE
YEARS IN THE AREA OF TRAUMATIC
BRAIN INJURY AND GREAT TO HAVE
YOU ON, TERRY.
FINALLY, ROY SILICA PREVENTION
OF PATHOLOGY AT IMMUNOLOGY AT
BAYLOR COLLEGE OF MEDICINE AND
PRIOR DIRECTOR OF THE T32
TRAINING PROGRAM THERE.
HIS LAB FOCUSES ON DETERMINING
THE BIOLOGICAL BASIS OF
UNDERLYING NEUROLOGIC DISEASES
PRIMARILY THOSE RELATED TO
DISEASE LINKED GENES AND RELATED
THE NANDS LANDER'S AWARD FOR
MENTORSHIP BY AN NANDS
INVESTIGATOR.
GOOD TO HAVE YOU HERE.
THOSE ARE THE NEW FOLKS.
>> I BELIEVE IT'S MY
RESPONSIBILITY NOW.
IT'S GREAT TO SEE EVERYONE NOW.
SO I'M JUST GOING TO RUN THROUGH
A SERIES OF HOUSEKEEPING ISSUES.
AS WALTER MENTIONED I WANT TO
CALL OUT JEANNE AND THE
COMMITTEE FOR STICKING AROUND
ANOTHER YEAR.
WE REALLY APPRECIATE IT.
DAN, FRANCIS, AMY, KATE, JEN,
ROY, YOU ARE SUBJECT MATTER
EXPERTS.
PLEASE, PLEASE WEIGH IN AND
PARTICIPATE ALL YOU CAN.
WE JUST CAN'T LET YOU VOTE AND
WE DO APPRECIATE ALL THE
PATIENTS RUNNING THROUGH THE
SLATE A3R0E68 PROCESS IN THE
FRONTCOURT.
THE SLATE APPROVAL PROCESS IN
THE OPEN SESSION.
WE'RE VIDEOCAST LIVE OUT TO THE
WORLD.
AND WE WILL HAVE A NUMBER OF
PEOPLE WHO ARE GOING TO BE ON
VIRTUALLY SO THAT'S WHY WE'RE
ASKING EVERYONE TO KEEP THEIR
CAMERAS ON TO ALL SEE OUR FACES.
NOW, AS SOME WILL RECALL WE
ATTEMPTED TO JOIN THE ELECTRONIC
COUNCIL BOOK FOR VOTING.
WE RAN INTO A NUMBER OF I CALL
THEM TECHNICAL CHALLENGES LAST
NIGHT.
WE TRIED TO GET SOME RESOLUTION
AND WHAT WE'LL DO THIS TIME IS
ATTEND THE OLD FASHION WAY OF
VOTING.
I'LL CALL FOR A MOTION AND WHEN
WE MAKE A MOTION TO BE SO MOVED
FOR THE PROPOSED PLAN OR WE CAN
MODIFY THAT IN THE MOTION AND
THEN ASK FOR A SECOND AND ASK BY
NAME YOUR VOTE.
WE'RE TALLYING THOSE AND HAVE TO
OFFICIALLY TALLY.
WE'RE NOT DOING IT TO BE
DRAMATIC BUT WANT TO KEEP GOOD
RECORDS HERE.
ON THE EDGE OF YOUR SEAT WAITING
FOR THAT VOTE.
SO THOSE ARE JUST THE OPENING
REMARKS.
ARE THERE ANY QUESTIONS?
I KNOW WE'RE HAVING TROUBLE WITH
THE ECB RIGHT NOW.
GINA?
>> I DON'T WANT TO QUESTION THE
METHOD.
I THINK IT'S FINE BUT WHEN IT
WAS ALL ZOOM WE JUST DID IT ON
ZOOM.
>> I'LL MAYBE PASS IT OVER TO MY
COLLEAGUE.
I THINK THEY FROWNED ON VOTING
IN THE ZOOM AND WE WANTED A HAND
COUNT.
WE DID LOOK INTO THAT.
>> WE WERE TOLD IT WAS NOT FACA
REGULATED TO DO IT THAT WAY.
I DID WANT TO ADD WHEREAS IF
YOU'RE IN PERSON YOU CAN RAISE
YOUR HAND.
IF YOU'RE ON ZOOM, WE DO NEED
YOU TO DO AN AUDIO YES, NO OR --
YEA, NAY.
>> WE'RE FOLLOWING THE RULES
BEST OF OUR ABILITY.
SO, ARE THERE ANY OTHER
QUESTIONS?
I MENTIONED WE'LL GET BOTH
THINGS SORTED OUT WITH THE
COUNCIL BOOK.
WITH THAT WE'RE MOVING TO OUR
FIRST ITEM WHICH WILL REQUIRE A
VOTE.
SO THIS WILL BE OUR BIG TEST
HERE.
WE'RE GOING TO CONSIDER THE
MINUTES.
THE MINUTES FROM THE SPRING '24
COUNCIL MEETING AND I HOPE
EVERYONE STUDIED THEM BEFORE
ATTENDING TODAY.
SO, DO I HAVE A MOTION ON THE
MINUTES?
DO I HAVE A SECOND?
OKAY.
HANK AND TIM, FIRST AND SECOND.
SO NOW I'M GOING TO CALL FOR THE
VOTE IN ORDER, AMY.
WE HAVE TWO AMIES.
WE NEED AMY A. AND AMY B.
AMY BRIN IS ON THE ZOOM.
>> GOOD MORNING.
APPROVE.
>> THERE'S ONE.
>> BOB.
>> APPROVE.
>> PERFECT.
JEN.
OKAY.
JANE.
THERE WE GO.
JIN MU.
>> APPROVE.
>> JOHN.
>> APPROVE.
>> LUIS.
HANK.
GINA?
>> APPROVE.
>> KIMIMI.
>> APPROVE.
>> TIM.
>> VERY GOOD.
SO LOOK AT THAT WE WERE ABLE TO
ACCOMPLISH THAT AND VERY GOOD.
SO MOVING TO OUR SECOND
HOUSEKEEPING ITEM IS DATES OF
FUTURE COUNCILS.
AGAIN THESE ARE LISTED AND WHAT
WE ASK YOU IS TO RESERVE THESE
ON YOUR CALENDAR FOR FUTURE
MEETINGS SO WE DON'T HAVE
CONFLICTS.
I DON'T THINK I'LL READ THEM ALL
OUT.
THEN WE GET TO OUR EXPEDITED
REVIEW PROCESS.
WHAT WE DO IS THIS IS WHEN THERE
ARE APPLICATIONS THAT ARE WELL
SCORED WITH NO ADMINISTRATIVE
ISSUES.
WE WORK TO GET THESE KIND OF
MOVING FORWARD WHAT WE HAVE IS
ASK THREE COUNCIL MEMBERS IF
THEY CAN VET THESE FOR US TO
MAKE SURE EVERYTHING'S KOSHER.
THIS TIME WE HAD JANE AND JIM
OUR READERS.
THANK YOU ALL FOR GOING OVER
THOSE AND TO PUT A NUMBER BEHIND
THAT WE HAVE 45 APPLICATIONS
THAT WERE EXPEDITED WE'RE IN THE
PROCESS OF ISSUING THESE NOW AND
THAT'S A TESTAMENT TO OUR GRANTS
MANAGEMENT BRANCH AND SHELLY
WILLBURN AND A WAY TO HOPEFULLY
GET MONEY IN RESEARCHERS' HANDS
SOONER RATHER THAN LATER.
NOW MOVING TO OUR EXTRAMURAL
ANNOUNCEMENTS.
I BELIEVE WE HAVE A SLIDE.
THIS IS THE VARIOUS COMINGS AND
GOINGS OF STAFF.
I'LL TAKE A MOMENT OF SILENCE.
WE CAN ALL TAKE A LOOK AT THAT.
WE CAN READ THE BIOS FOR EACH OF
THESE AND YOU CAN IMAGINE IT CAN
TAKE A WHILE.
I CO KNOW WHAT WE DO HAVE IS
WITH HAVE TWO LONG STANDING
MEMBERS OF OUR STAFF WHO WILL BE
RETIRED.
I DO WANT TO PASS IT TO LYNN
JAKEMAN TO DO THE HONOR OF THESE
ANNOUNCEMENTS.
>> THANK YOU ALL.
I APPRECIATE TAKE A COUPLE
MINUTES JUST TO HIGHLIGHT A
COUPLE LONG-TERM PROGRAM
DIRECTORS REACHING RETIREMENT
THIS YEAR.
GIVE AN OPPORTUNITY TO RECAP THE
WORK THEY'VE DONE.
THE FIRST OF THESE IS DR. JIM
KENIG AND HE JOINED NANDS IN
2015 AS A TRUE PHYSIOLOGIST
THROUGH AND THROUGH.
HE GOT HIS BACHELOR'S AT THE
UNIVERSITY OF BIRMINGHAM AND HAD
AN ILLUSTRIOUS CAREER BEFORE
JOINING.
HE CAN WEAR THE CROWN FOR HAVING
THE LARGEST MOST DIVERSE IN
TERMS OF MECHANISMS AND GRANT
PORTFOLIO FOR THE LAST SELF
YEARS.
HE HAS MANAGED THE GRANT
PORTFOLIO IN ISCHEMIC STROKE.
HE ALSO WAS A LONG-TERM POINT OF
CONTACT FOR THE HERBAL POLICY
PROGRAM AND CONTRIBUTED TO THE
STRATEGIC PLANNING FOR CEREBRAL
PALSY AND CARRIED LONG BEFORE IT
IS AS BIG AS IT IS NOW WORK ON
WOMEN'S HEALTH BY ENCOURAGING
INVESTIGATORS TO INCLUDE SEX AS
A BIOLOGICAL VARIABLE IN THEIR
RESEARCH.
HE WAS THE NANDS POINT WHEN SOME
OF THAT WAS ROLLED OUT.
HE'S BEEN AN OUTSTANDING CITIZEN
IN EVERY SINGLE WAY.
HE'S BEEN HERE IT WILL BE 12
YEARS IN DECEMBER AND HAS BEEN
OUTSTANDING PROGRAM DIRECTOR.
I WANTED TO GIVE HIM A MOMENT OF
DUE AND THANK YOU, JIM.
THE SECOND PROGRAM DIRECTOR WHO
IS RETIRING AT THE END OF THE
YEAR IS DR. JIM KNOTT AND
STARTED IN 2008 LONG BEFORE I
DID.
HE GOT HIS DEGREE IN PHYSIOLOGY
AND BIO PHYSICS BUT HIS
SPECIALTY AS A PROGRAM DIRECTOR
AT NANDS HAS BEEN IN NEURAL
CIRCUITS.
HE WORKED AT SOME PERIOD AT
HOWARD UNIVERSITY AND WAS A BIG
PART OF OUR DIVERSITY PROGRAMS
GETTING THEM OFF THE GROUND IN
THE EARLY DAYS AS PART OF THE
DIVERSITY WORKING GROUP.
HE ALSO HAD EXPERIENCE WORKING
WITH LAND ANIMALS AND HE HAS
BEEN A POINT OF CONTACT WITH
HELPING US TO NAVIGATE SOME OF
THE TRICKY ISSUES WITH THE USE
OF LIVE ANIMALS AND NON-HUMAN
PRIMATES.
AND IN 2013 I'LL TELL ONE LITTLE
STORY BECAUSE IT'S NOT THAT
LITTLE.
WHEN RUMORS WERE ABOUNDING THE
PRESIDENT HAD A BIG INITIATIVE
UP HIS SLEEVE, THERE WERE A
GROUP OF STAFF PRIMARILY FROM
NANDS AND NIMH WHO WORKED
TOGETHER IN THE BEGINNING
OCTOBER 1st THERE WAS A SHUTDOWN
AND THEY COULDN'T WORK BECAUSE
YOU CAN'T WORK DURING A SHUT
DOWN BUT HAD LUNCHES AND DINNERS
AND CAME BACK WITH THE CONCEPT
OF THE BRAIN INITIATIVE AND WAS
ENTIRELY STAFF DRIVEN IN THE
INITIAL STAGES AND HE TOOK ON
THE CIRCUITS AND SYSTEMS
COMPONENT OF TRYING TO FIGURE
OUT WHAT THE BRAIN INITIATIVE
COULD LOOK LIKE BY 2025.
I'D LIKE TO THANK HIM AS AN
ACCOMPLISHED PROGRAM DIRECTOR.
>> THANKS, LYNNE.
JIM AND JIM, CONGRATULATIONS AND
DO NOT GO GENTLY IN THE NIGHT.
WITH THAT, WALTER, I'LL PASS IT
BACK TO YOU.
>> THANKS, DAVE.
I'LL EMPHASIZE THERE'S A
TREMENDOUS AMOUNT OF WORK THAT
GOES ON DAILY HERE AT NANDS,
STAFF ARE DEEPLY COMMITTED TO
THE MISSION OF KNOW INSTITUTE
AND DEEPLY COMMITTED TO HELPING
INVESTIGATORS BE SUCCESSFUL IN
ADVANCING OUR MISSION BY
ADVANCING THEIR RESEARCH.
I'M PROUD OF THE PEOPLE WHO WORK
HERE.
I WISH WE COULD HIGHLIGHT
EVERYBODY BEFORE THEY RETIRE.
A LOT OF PEOPLE DON'T SURVIVE.
I'LL GIVE ME UPDATE TO THE
COUNCIL MY DIRECTOR'S REPORT.
>> FIRST I WANTED TO MENTION
THAT THERE IS A NEW PERSON IN
THE LEADERSHIP ROLE AT THING OFF
OF STRATEGIC COORDINATION.
THIS IS IN THE BUILDING 1,
DR. BERTAGNOLLI'S OFFICE AND
DR. CAROLYN HUTTER TO RUN THE
OFFICE OF STRATEGIC COORDINATION
WHICH RUNS A NUMBER OF THINGS
BUT MOST IMPORTANTLY THE COMMON
FUND PROJECTS.
THAT'S AROUND $700 MILLION.
IT GOES TO THE OFFICE OF THE
DIRECTOR TO DO RESEARCH THAT
CUTS ACROSS INSTITUTES AND TRIES
TO DO RESEARCH THAT'S FORWARD
LOOKING.
HERE AT OUR INSTITUTE WE'VE
ALWAYS BEEN ACTIVE IN TRYING TO
GET ENGAGED IN THESE PROJECTS
BECAUSE WE WANT TO BRING THOSE
NEW SCIENCE TO NEUROSCIENCE.
SO SOME OF THOSE INCLUDE THE
ACUTE TO CHRONIC PAIN SIGNATURE
PROGRAM ONGOING NOW RUN BY OUR
OFFICE OF PAIN, POLICY AND
PLANNING.
THE ALS TRANSFORMATIVE PROJECT.
THE FACULTY INSTITUTION
RECRUITMENT FOR SUSTAINABLE
TRANSFORMATION WHICH IS A
CO-HORT HIRING PROGRAM TO HIRE
FOLKS INTO DEPARTMENTS AND
UNIVERSITIES WHERE THEY WOULD BE
VERY UNDER REPRESENTED BY
TOGETHER THE IDEA WOULD BE
THEY'D HAVE A MORE CHANCE OF
BEING SUCCESSFUL WORKING WITH
EACH OTHER AS THEY MOVE INTO THE
ACADEMIC CAREERS.
IT'S AN IDEA WE HAD YEARS AGO
AND WE'RE WORKING ON PUTTING IN
AND COMMON FUND THOUGHT IT WAS
GOOD AND TOOK IT WHICH IS GREAT
BECAUSE IT'S THEIR MONEY.
AND THE SOMATIC CELL GENE
EDITING PROGRAM WAS AN EXCITING
PROGRAM ONGOING NOW IN THE
SECOND PHASE.
THE FIRST PHASE WAS DEVELOPING
TOOLS TO BE ABLE TO GET PEOPLE
TO DO GENE EDITING IN VIVO AND
NOW IN THE SECOND PHASE WE'RE
FUNDING PROJECTS TO DO GENE
EDITING IN VIVO.
WE'LL HEAR MORE LATER ON BUT
CLEARLY IT WILL TREAT
NEUROGENETIC DISORDERS BUT
POTENTIALLY CAN DO THIS SAFELY
THE GENE EDITING MAY BE VERY
IMPORTANT FOR MORE COMMON
DISORDERS, CHANGING VARIANTS,
DECREASING THE RISK OF
NEUROLOGIC DISEASES.
THERE'S THE SPARK PROGRAM WHICH
WAS LOOKING AT INNOVATION IN THE
BODY THROUGH THE AUTONOMIC
SYSTEM AND THERE'S ANOTHER
PROJECT TRYING TO DEVELOP AN
ATLAS OF SOMATIC MUTATIONS.
AS PEOPLE KNOW THESE HAVE BEEN
FOUND TO BE IMPORTANT IN CAUSING
TROUBLE WITH NEURODEVELOPMENT IN
EPILEPSY.
THERE'S A NEW PROGRAM THAT WE'RE
JUST WORKING ON NOW.
IT'S MEDICAL IMAGING, BRINGING
MEDICAL IMAGING WITH OTHER
HEALTH DATA INTO A SYSTEM WHERE
A.I. ALGORITHMS CAN BE USED TO
MAKE PREDICTS THAT WOULD BE
VALUABLE IN PREDICTING ISSUES IN
SOMEONE'S HEALTH.
THERE'S A LOT OF A.I. GOING ON
IN THE HEALTH BUSINESS BUT
SURPRISINGLY NO ONE HAS BEEN
ABLE TO ACTUALLY OVERCOME THIS
BARE SO THERE'S BIOLOGICAL DATA
SITTING IN IMAGES AND
HOSPITALING AROUND THE COUNTRY
THAT HAVE NOT BEEN ABLE TO BE
ANALYZED AT SCALE AND THAT'S A
HOPE OF THAT PROGRAM.
AND 80% HAD NEURO LONLIC
CONDITIONS AND WHEN THAT COMMON
FUND PROJECT NANDS TOOK THE LEAD
IN CONTINUING THAT WORK AND WILL
CONTINUE IN THE FUTURE.
THE NEXT SLIDE IS TO POINT OUT
SOME OF THE PRIORITIES FROM
DR. BERTAGNOLLI, THE NIH
DIRECTOR.
THE EDITORIAL IS TALKING ABOUT
HER TWO MAJOR PRIORITIES TO
BRING RESEARCH INTO THE
COMMUNITIES, PRIMARILY BY
PRIMARY CARE SETTINGS AND THE
ISSUE I THINK IS THERE TO BE
TAKEN IS THE FACT THAT OUR
HEALTH IN THE U.S. GIVEN THE
DOLLARS WE SPEND RATES VERY LOW
COMPARED TO OTHER COUNTRIES AND
ONE OF THE REASONS IS BECAUSE
OUR PREVENTATIVE HEALTH AND
RESEARCH HAS REALLY NOT STOOD UP
TO THE RESEARCH WE DO FOR WHEN
YOU GET SICK.
SO THE ONLY WAY TO REALLY SOLVE
THAT IS TO WORK AT THE COMMUNITY
LEVEL IN THE PRIMARY CARE
SETTING WHICH IS SOMETHING THAT
HASN'T BEEN DONE VERY
SUCCESSFULLY IN THE PAST.
IT'S A BIG CHALLENGE.
THAT'S WHAT MONICA IS HOPING TO
DO.
THE SECOND EDITORIAL I THINK WAS
IN SCIENCE AND IT WAS JUST
POINTING OUT ONCE AGAIN NIH'S
SUPPORT FOR ASIAN IMMIGRANT AND
ASIAN RESEARCH COLLEAGUES
ACKNOWLEDGING THE FACT THAT THE
ISSUES THAT OCCURRED WHERE
PEOPLE'S SUPPORT WAS SCRUTINIZED
AND PEOPLE WERE FOUND TO HAVE
SUPPORT FROM OTHER PLACES THEY
DIDN'T ACKNOWLEDGE ON THE GRANTS
LED TO A LOT OF TROUBLES AND
BOILED OVER ASIAN SCIENTISTS
TOOK THE BRUNT OF THAT
DISCRIMINATION AND THE GOOD NEWS
IS THOSE INCIDENTS NOW ARE
BASICALLY A TRICKLE IF FIG.
PEOPLE HAVE LEARNED TO BE
TRANSPARENT WITH SUPPORT BUT
STILL I THINK MONICA'S COMING
OUT IN SCIENCE WITH THIS BACKING
OF THE ASIAN AMERICAN SCIENTISTS
TO GET THINGS BACK TO NORMAL AND
CERTAINLY FOR US, 20% OF OUR
GRANTEES ARE ASIAN AMERICANS.
THEY'RE MAJOR CONTRIBUTORS TO
OUR MISSION AND TREMENDOUSLY
VALUED HERE AT NANDS.
A COUPLE NEW THINGS PEOPLE
COMING ON YOU MAY RECALL NINA
SHORE LEFT TO BECOME THE ACT
BEING SCIENTIFIC DIRECTOR IN THE
INTRAMURAL PROGRAM AND HIRED TO
BE HEAD OF INTRAMURAL AT NIH AND
AMY ADAMS HAS TAKEN OVER AND
SHE'S EFFICIENT AND ADEPT IN HER
LEADERSHIP ROLE.
WE HAVE WHAT'S CALLED A SELECTED
OFFICIAL.
THEY HAVEN'T REALLY GOTTEN THEIR
NOTICE THEY'RE ON PROBATION
WHICH IS WHEN YOU COME TO NIH
YOU GET A LETTER SAYING YOU'RE
ON PROBATION FOR A YEAR AND I
REMEMBER I GOT ONE AND I THOUGHT
I HADN'T DONE ANYTHING WRONG
YET.
WE'RE HOPING ANNAPURNA WILL COME
ON AND SHE'S A PEDIATRIC
NEUROLOGIST FOCUSSED ON
UNDERSTANDING THE CAUSES OF
HUMAN EPILEPSY.
MANY CONDITIONS THAT FALL UNDER
THE GENOMIC THERAPIES WOULD BE
EPILEPSIES AND CLOSE TO
RECRUITING A NEW DIRECTOR OF
EXTRAMURAL ACTIVITIES AND THAT'S
ANDREA MEREDITH A PROFESSOR AT
UNIVERSITY OF MARYLAND AND HER
RESEARCH OVER THE YEARS HAS BEEN
FOCUSSED MOSTLY ON BK CHANNELS
AND IN THE BIOPHYSICAL SPACE AND
WAS GETTING FROM DIFFERENT
INSTITUTES AT NIH DEPENDING ON
THE ORGAN SYSTEM.
PRIMARILY A BIOPHYSICIST WHO RAN
INTO A MUTATION THAT CAUTIONS
ULTRA RARE AMNESIA AND
DYSKINESIA DISORDER AND THAT
DISEASE AND LOOKING FORWARD TO
ANDREA COMING IN.
GREAT THANKS TO DR. DAVID OWENS
AS THE ACTING DEA DIRECTOR.
I WE HAVEN'T MISSED A STEP AND
SHE'S MOVED TO BECOME THE
DIRECTOR AT NIBIB.
WE'LL MISS ANNA BUT WE KNOW
WHERE SHE LIVES.
NOW, TO SOME SOBERING NEWS.
WE'RE ALWAYS VERY TRANSPARENT IN
TERMS OF TALKING TO THE COUNCIL
AND TO THE PUBLIC ABOUT OUR
FINANCIAL SITUATION AND OUR
BUDGET.
SO, WHAT SHOW SLIDE SHOWS IN
GREEN ARE PAST YEARS AND AS YOU
PLAY KNOW WE WERE FLAT BETWEEN
2004 AND 2016 WITHOUT MUCH
INCREASE AT ALL BUT IN 2016 WE
STARTED TO GET YEARLY INCREASES
WHICH REALLY HELPED US TO DO A
GREAT MORE SCIENCE AND FUND A
LARGER NUMBER OF PEOPLE INTO
NANDS AND THE DIFFICULTY AS YOU
DO THAT IS YOU FUND MORE GRANTS,
YOU INCUR MORE OUT YEARS AND IF
YOU DON'T GET AN INCREASE THAT
AT LEAST KEEPS PACE WITH
INFLATION YOU RUN INTO WHAT IS
CALLED A CLIFF WHICH IS WHERE WE
WENT OVER IN '24 AND STILL OVER
THAT CLIFF.
IN '24 SEE THE TOTAL
APPROPRIATION DROPPED BY 4.4%
LARGELY DUE TO THE DECREASE IN
FUNDING TO THIS BRAIN INITIATIVE
OTHERWISE THINGS WERE PRETTY
FLAT AND THE COST OF GRANTS WENT
UP.
THAT CAUSED US NOT TO BE ABLE TO
FUND AS MANY GRANTS AS WE WISH
AND THEN FORCED US TO DO THINGS
AND GARNER MONEY AND TRIED TO
PUT A FORCE ON THE COST FOR
GRANT WE'LL GET TO IN A SECOND.
LOOKING FORWARD TO 2025, WHICH
FOR US STARTS OCTOBER 1 WE'RE
COMING TO THE END OF THE YEAR.
A NEW CAREER STARTS OCTOBER 1.
LOOKING FORWARD TO 2025, WE HAVE
BASICALLY THREE BUDGET NUMBERS
TO LOOK AT ONE IS THE
PRESIDENT'S BUDGET WHICH
RESTORES THE BRAIN INITIATIVE,
GIVES US A TOTAL OF 5.3%
INCREASE BUT DOES NOT INCREASE
THE FUNDS FOR REGULAR GRANTS.
THE HOUSE DOES NOT RESTORE THE
BRAIN INITIATIVE AND THE SFLAT
DOES RESTORE THE BRAIN
INITIATIVE MONEY.
WE'LL TALK IN DETAIL ABOUT SOME
OF THESE NUMBERS.
I WANT TO START BY SHOWING THE
TOP LINE WHICH ENCASED IN RED.
THE FUNDS AVAILABLE FOR WHAT WE
CALL NORMAL PROCESSES.
NOT TARGETED FUNDS.
THIS IS THE FUND IING FOR RPG
FUNDING MAKING UP THE VAST
MAJORITY.
WE INCREASED FROM 2020 FROM
$1.97 MILLION TO 2023 TO $2.167
MILLION AND THEN IN '24 WAS
FLAT.
IN '25 THE PRESIDENT'S BUDGET IS
PRETTY FLAT.
THE HOUSE MARK IS A LITTLE BIT
INCREASED THE SENATE MARK IS
FLAT.
WE HAVE TO PREPARE OUR A FLAT
BUDGET AT LEAST THAT'S OUR BEST
SITUATION TO GET A FLAT BUDGET
PUTTING US IN THE SAME SPOT WE
WERE IN LAST YEAR.
THE EXPECTATION IS HAVING A
CONTINUING RESOLUTION YOU
OPERATE AS IF YOU HAD THE SAME
AMOUNT YOU HAD LAST YEAR BUT
THAT'S A GUESS AND YOU MAY HAVE
OVERSPENT OR UNDER SPENT AND YOU
WANT TO UNDER SPEND BECAUSE IF
YOU OVER SPEND YOU HAVE AN
IMBALANCE WHERE YOU FUNDED
GRANTS AND HAVE TO STOP HALFWAY
THROUGH THE YEAR.
WE ACT CONSERVATIVE AND FIGURE
WE'LL GET THROUGH IT OKAY BUT WE
CAN ALWAYS INCREASE THE PAY LINE
WHEN WE HAVE A BUDGET AND THEN
GO BACK AND PAY GRANTS WE DIDN'T
FUND SAY THIS COUNCIL CAN FUND
THEM IN FEBRUARY IF WE HAVE A
BUDGET OR MAY IF IT TAKES THAT
LONG TO HAVE A BUDGET.
GIVEN AN ELECTION YEAR AND THEY
AGREED ON A FLAT BUDGET FOR '24
WHICH WE GOT AND ALSO AGREED THE
'25 BUDGET WOULD NOT BE MORE
THAN 1% INCREASE OVER THE '24
BUDGET.
THAT PUTS US IN A ROIF
SITUATION.
THE NEXT ROW IS THE BRAIN
INITIATIVE.
IN 2020 THE BRAIN INITIATIVE HAS
TWO BUCKETS OF MONEY.
O
ONE, THE BRAIN AND 21st
CENTURIES CURES BUDGET AND THEY
HAD AN AMOUNT FROM CURES AND
70 -- $70 MILLION FROM CURES AND
$28.9 MILLION FROM THE BASE OF
NINDS.
MI MH IS GETTING THE SAME THING
WE'RE GETTING.
IF YOU FOLLOW THE NUMBERS ACROSS
YOU CAN SEE THE CURES MOANY GOES
DOWN AND THEN UP AND THEN IN
2023 IT GOES FROM $76 MILLION TO
$225 MILLION.
WHAT HAPPENED THERE WAS THE
FOLKS AT THE NIH BUDGET SAID
THIS IS A BIG INCREASE AND WE
CAN'T GIVE YOU ALL THAT CURES
MONEY ON TOP OF THE BASE WE'RE
GOING TO TAKE THE CURES MONEY
BUT REDUCE THE BASE.
IN THE IDEA WAS IN '24 WHEN THE
CURES MONEY GOES DOWN TO $86
MILLION THEN THE EXPECTATION WAS
THEY RAISE THE BASE AGAIN BUT
THEY DID NOT DO THAT.
THAT CAUSED THIS 40% DROP IN THE
BRAIN INITIATIVE BUDGET IN 2024.
IN 2025 YOU CAN SEE THE CURES
MONEY DROPS AGAIN ALMOST IN HALF
AND THE BRAIN BASE BUDGET IS
REALLY QUESTIONABLE WHAT'S GOING
TO HAPPEN THERE.
THE PRESIDENT'S BUDGET RESTORES
THE BASE, $243 MILLION AND THE
SENATE MARK DOES THE SAME BUT
THE HOUSE MARK KEEPS IT FLAT.
THE BASE BUDGET IS WHAT YOU RELY
UPON IN ANY KIND OF LONG-TERM
MONEY.
THE CURES MONEY DISAPPEARS
COMPLETELY IN 2026.
MOVING ALL THESE ADVANCES FROM
THE BRAIN INITIATIVE FORWARD
RELIES ON GETTING THE BASE
BUDGET BACK.
FOR NINDS I'D SAY 90% OF THIS
PEOPLE FUNDED BY BRAIN ARE GOING
TO BE COMING TO NINDS IF THERE'S
NO BRAIN MONEY PUTTING INCREASED
PRESSURE ON NINDS IN TERMS OF
FUNDING AND THEY'RE GOOD
PROJECTING SO IT WILL MAKE
THINGS REALLY COMPETITIVE IF
THAT HAPPENS.
THAT'S THE STORY WITH THE BRAIN
BUDGET.
JOHN NGAI IS HE DIRECTOR AND HIS
ABILITY COULD BE REDUCED.
THE PLAN WITH BRAIN IS TO TAKE
IN THE GRANTS, WE PRIORITIZE ALL
THE GRANTS AND BRING THEM TO THE
MULTI-DISCIPLINARY WORKING GROUP
BUT WE'RE NOT GOING TO FUND
ANYTHING UNTIL WE KNOW WHAT THE
BUDGET IS.
WE'RE JUST GOING TO SIT AND HAVE
THESE THINGS TALLIED UP AND ONCE
WE KNOW HOW MUCH MONEY WE HAVE
WE CAN START FUNDING THE GRANTS
BECAUSE THERE'LL BE GRANTS
COMING IN THROUGHOUT THE YEAR IF
YOU SPENT ALL YOUR 30 NOW YOU
CAN'T FUND ANYTHING ELSE THE
REST OF YEAR.
THE THOUGHT IS WHAT COMES IN
THROUGH THE YEAR AND HOW MUCH
MONEY WE HAVE AND THEN MAKE THE
DECISION ON FUNDING.
THERE ARE OTHER CONGRESSIONALLY
MANDATED FUND.
THE HEAL INITIATIVE, WHICH WE'LL
TALK ABOUT IN A SECOND.
DOWN THE ROAD IS HELPING TO END
ADDICTION LONG TERM.
MONEY GOES TO THE NATIONAL
INSTITUTE OF DRUG ABUSE
PRIMARILY TO REDUCE THE DAMAGE
TO THE COUNTRY DUE TO OPIOID
OVERDOSES AND DEATHS WHICH ARE
STILL TRAGICALLY HIGH.
FUNDS ALSO COME TO NINDS AND WE
WORK WITH 14 INSTITUTES THAT
HAVE PAIN PORTFOLIOS TRYING TO
DEVELOP NON-ADDICTIVE
THERAPEUTICS OR NON-ADDICTIVE
STRATEGIES FOR PAIN.
IT'S BEEN WILDLY SUCCESSFUL AND
THIS IS FUNDING RIGHT NOW IT'S
AT THE BASE AND LOOKS LIKE
SOMETHING WE CAN COUNT ON GOING
FORWARD AND WE'LL BE TALKING
ABOUT STRATEGIC PLANNING COMING
UP FOR THIS PURPOSE.
CONGRESS ALSO GIVES US FUNDS FOR
ALZHEIMER'S RELATED DEMENTIA
RESEARCH.
CONGRESS IS INCREASED THE
FUNDING FOR DEMENTIA RESEARCH
FOR ABOUT $3.5 MILLION OVER THE
LAST FIVE OFFER SIX YEARS.
WE MARCHING ABOUT 10% OF THAT
MON -- WE MANAGE ABOUT 10% OF
THAT MONEY.
INITIALLY IT ALL WENT TO THE
AGING INSTITUTE AND WE WORK WITH
THE AGING INSTITUTE AND
BASICALLY ALL THE MONEY'S IN THE
POT AND WE USE ABOUT 10% OF IT
CURRENTLY AND THE ALZHEIMER'S
RELATED DEMENTIA ARE FRONTAL
TEMPORAL AND VASCULAR DEMENTIA
AND LEWY BODY AND PARKINSON'S
DISEASE AND WE WILL HEAR FROM
OTHERS LATER ON SUMMARY OF
WHAT'S GOING ON IN THAT SPACE.
I MENTIONED THE COMMON FUND
MOVED THE UNDIAGNOSED DISEASE
NETWORK TO NINDS IN 2023 WITH A
BUDGET OF ABOUT $18 MILLION.
WE CUT ABOUT $4 MILLION FROM THE
INSTITUTE.
THE NINDS BUILDING 1 HAS BEEN
GIVING US THE DIFFERENCE TO MAKE
UP $18 MILLION BUT WE UNDERSTAND
THAT'S GOING TO STOP.
THIS IS THE LAST YEAR FOR THAT.
NEXT YEAR THE HOPE IS WE GET AN
APPROPRIATION FOR THE $18
MILLION.
THE APPROPRIATION NOW IS ONLY $2
MILLION.
GINA.
>> I'M CURIOUS WHETHER THERE'S A
NEW CALL OUT FOR ALZHEIMER'S
DISEASE FOR THE ONE TOX NETWORK.
IS THAT PART OF THIS BUDGET OR
IS IT A NEW INITIATIVE?
>> GOOD POINT.
I SHOULD HAVE MAY BE TALKED MORE
ABOUT THAT BUT TO QUALIFY FOR
ALZHEIMER'S RELATED DEMENTIA
WITH A DIFFERENT PAY LINE
BECAUSE THERE'S MORE MONEY
THERE, EITHER YOU COME TO AN RFA
PUT OUT UNDER ALZHEIMER'S
RELATED DIM OWN THAT WHICH IS
LOOKING AT ANY KIND OF
ENVIRONMENTAL INFLUENCES ON
DEVELOPING NEURODEGENERATION
WOULD FIT INTO BUT ALSO ANY
GRANT THAT COMES IN HAS AN
ALGORITHM THAT LOOKS AT THE
SPECIFIC AIMS AND ABSTRACT AND
CODES THEM AS ALZHEIMER RELATE
ED -- RELATED DEMENTIA AND GO
TO THE FUNDING WE HAVE AND WE
MAINTAIN THE SAME PAY LINE AS
THE AGING INSTITUTE.
PEOPLE CAN COME IN WITH
ALZHEIMER'S RESEARCH OR OTHER
DEMENTIA RESEARCH, BOTH
INSTITUTES OR THE EFFECTS ON
MAYBE SLEEP DISTURBANCES ON
NEURODEGENERATION FOR INSTITUTE
TO GET THE SAME PAY LINE.
THE LAST THING IS THE SENATE IN
THE 2025 MARK BUT $25 MILLION
FOR ALS RESEARCH SEPARATE FROM
ADDITIONAL MONIES WHICH GO TO
THE OFFICE OF THE DIRECTOR AND
COMES TO US FOR THE ACT FOR ALS
AND THAT'S $75 MILLION
SUPPORTING A PUBLIC-PRIVATE
PARTNERSHIP WITH MULTIPLE
COMPANIES AND NON-PROFS
ORGANIZAT
ORGANIZATIONS TO HOOK FOR
TARGETS IN BIOMARKERS TO DEVELOP
TREATMENT FOR ALS AND WE'RE ABLE
TO FUND A CONSORTIUM AROUND THE
COUNTRY TO COLLECT CLINICAL DATA
AND SAMPLES WITH FOLKS FROM ALS
AS PART OF THE PUBLIC-PRIVATE
PARTNERSHIP.
I MENTIONED WE MANAGE ABOUT $309
MILLION IN TOTAL FOR ADRD
FUNDING.
THAT PUTS OUR.
WE MANAGE PRETTY MUCH $3 BILLION
A YEAR OF FUNDING.
I GUESS WHEN YOU THINK ABOUT THE
NEXT SLIDE YOU HAVE TO REMEMBER
IT'S $3 BILLION A YEAR.
THIS IS IN PREPARATION FOR THE
NEXT SLIDE WE TALK ABOUT THE PAY
LINE.
SO GIVEN THE BUDGETARY
CONDITIONS WE'VE HAD IN '24 AND
NOW LOOKING TO '25.
WHAT WE'D LIKE TO DO AS BEST WE
CAN IS PROTECT THE PAY LINE.
AND I MUST SAY WE HAVEN'T BEEN
ABLE TO DO THAT VERY WELL.
THERE'S NO OTHER OPTION BECAUSE
WE HAVE A NUMBER OF GRANTS THAT
RUN OUT OF MONEY AND THE COST OF
THE GRANTS WENT UP SO WE'RE DOWN
TO CONSERVATIVE ESTIMATE
STARTING OUT THE YEAR WITHOUT A
BUDGET AT EIGHT PERCENTILE
SOMETHING I NEVER WANTED TO GET
CLOSE TO.
HOPEFULLY WHEN WE GET THE FY25
BUDGET WE GET A SENSE OF HOW
MANY GRANTS ARE COMING IN AND
HOW MUCH THEY COST AND BE ABLE
TO MOVE THE PAY LINE UP.
NOT SURE HOW FAR WE'LL BE ABLE
TO GET.
>> I JUST WANTED TO ASK WHETHER
THE TRAINING OR PROFESSIONAL
DEVELOPMENT COMES FROM THE NINDS
BASE AND IS THAT PROTECTED?
WHEN THAT GOES UP AND DOWN IS IT
PROTECTED?
>> GOOD POINT.
WE'RE THINKING THAT THROUGH.
WE HAVE A SLIDE ON THAT.
DISCUSSION WITH THE COUNCIL IN
THE PAST ARE PHILOSOPHY IS IN
ROUGH TIMES WE LIKE TO FUND AS
MANY INVESTIGATORS AS POSSIBLE
PARTICULARLY VULNERABLE
INVESTIGATORS WHO USE BRIDGE
AWARDS TO FUND PEOPLE WHO HAVE A
GOOD SCORE, THEY JUST MISSED AND
THEY DON'T HAVE ANY OTHER
GRANTS.
WE DID QUITE A BIT OF THAT THIS
YEAR AND WOULD BE ABLE TO HOLD
MONEY FOR NEXT YEAR.
THAT'S ONE OF OUR BUFFER FUNDS.
WE HAVE TO HOLD OFF UNTIL WE
KNOW EXACTLY WHERE WE'LL MAKE
OUR BUDGET AT THE END OF THE
YEAR AND THEN WHEN WE HAVE A
BETTER EXACT SENSE WE CAN START
RELEASING THOSE KINDS OF AWARDS.
WE LIKE TO GET TO THE END OF THE
BUDGET AND RETURN ABOUT 10 CENTS
TO THE TREASURY ON $3 BILLION.
WE GET UNDER 10 CENT S.
WE HAVE GOOD PEOPLE WORKING ON
IT AND GET CLOSE.
THE OTHER WHICH IS WHAT GINA
JUST BROUGHT UP IS WE WANT TO
UPHOLD A COMMITMENT OF FUTURE
HEALTH IN NEUROSCIENCE RESEARCH.
WE CAN'T MORTGAGE OUR FUTURE AND
WE ARE TRYING TO REALLY PROTECT
AS BEST WE CAN THE TRAINING
PROGRAMS AND THE CAREER
DEVELOPMENT PROGRAMS.
AND SO AGAIN WE ALSO REALIZE
THAT NINDS BECAUSE IT'S 100%
NEUROSCIENCE.
THE ONLY INSTITUTE THAT'S 100%
NEUROSCIENCE WE HAVE THIS
RESPONSIBILITY TO REALLY LEAD IN
NEUROSCIENCE HERE PARTICULARLY
IN THE AREA OF FUNDAMENTAL
NEUROSCIENCE RESEARCH.
EVERYTHING BUILDS OFF THAT AND
WE WANT TO CONTINUE TO SUPPORT
THOSE COMMITMENTS.
WE HAVE IN THE RECENT PAST
WORKED AND RICHARD BENZ WILL
TALK MORE TO DEVELOP PROGRAMS
THAT CAN MAKE A DIFFERENCE IN
PEOPLE SUFFERING WITH NEUROLOGIC
DISEASE AND NOT GETTING THE CARE
THEY SHOULD BE GETTING BECAUSE
OF WHERE THEY LIVE OR WHO THEY
ARE.
THIS RESEARCH IN LIMITING HEALTH
DISPARITIES WE NEED TO PROTECT
MOVING FORWARD.
WE LOOK TO OUR PORTFOLIO AND
DIDN'T FIND THINGS THAT SAYS
THIS IS NOT WORTH DOING AND TAKE
THE WHOLE CHURCHING OUT.
EVERYTHING IS REDUCED.
EVERYTHING GOT A HAIRCUT.
THIS IS WHAT THE PAY LINE LOOKS
LIKE FOR RO1s.
IN 2024 WE WENT TO THE SPLIT PAY
LINE.
THE IDEA IF SOMEONE HAS AN
EXPENSIVE GRANT DOING GREAT
SCIENCE WE'LL PAY IT AND SAME
FOR THE SCR REVIEW IF THEY COME
IN WITH A GRANT, IT'S REALLY
GREAT, WE'LL PAY IT BUT IT'S
REALLY GOT TO GET THAT HALF THE
REGULAR PAY LINE OR BETTER.
WE HAVE THAT SPLIT PAY LINE.
IN '23 WE HAD A 14 PERCENTILE
FOR EVERYBODY AND IN '24 DROPPED
TO 11 PERCENTILE.
THE LESS THAN 500K DIRECT COST
GRANTS HALF WAS 5% OR GREATER
FOR GREATER THAN 500K DIRECT
COST GRANTS.
IN 2025 THE INTERPAY LINE GOING
CONSERVATIVE BECAUSE WE HAVE TO
WE DROPPED TO 8% SO FOR GREATER
THAN 500K, 4% AND FOR EARLY
STAGE INVESTIGATORS IN '23 WAS
25%.
IN '24 WAS 22 WHICH IS DOUBLE
THE REGULAR AND IN '25 AGAIN
WE'LL DOUBLE THE REGULAR TO 16
PERCENTILE.
THE APPLICATION SUBMITTED TO
COUNCIL REVIEW ARE THOSE I
MENTIONED WHERE THE INVESTIGATOR
HAS OVER $1.5 MILLION IN TOTAL
COST FROM NIH GRANTS AND NEED TO
GET A BETTER PERCENTILE.
THAT'S OUR POLICY GOING FORWARD
AND WILL PUT IT ON THE WEBSITE.
IN TERMS OF THE NIA PAY LINE I
DON'T THINK IT'S PUBLIC YET.
DOES ANYBODY WANT TO CORRECT ME?
>> WHEN WE HEAR FROM NIA WE'LL
FUT OUR DATA FOR ALZHEIMER'S
RELATED DEMENTIA.
THIS IS THE ISSUE THAT IS THE
AVERAGE COST OF AN RO1 GRANT
FROM 2005 TO 2024.
IN THE PAST COUPLE YEARS SINCE
2020 THINGS SKYROCKETED RELATED
TO INFLATION.
IF YOU LOOK AT THE BOTTOM GRAPH
WHICH IS CORRECTED FOR INFLATION
THERE'S NO DIFFERENCE, PRETTY
FLAT.
IT'S NOT THE PEOPLE WILL GREEDY.
IT'S COSTS MORE.
THAT'S JUST THE ISSUE.
IF WE DON'T GET AN INCREASE TO
MIRROR THAT, YOU DON'T HAVE THE
MONEY TO FUND THE SAME NUMBER OF
GRANTS.
THAT'S THE SPOT WE'RE IN.
>> WALTER, I HAVE A QUESTION
BETWEEN THE AVERAGE MODULAR AND
REAL COST OF THE GRANTS.
HAS ANYONE ANALYZED WHERE THE
MODULAR DOESN'T EXIST ANYMORE?
I'M SURE SOMEONE HAS -- WHY'S IT
EXIST.
IT'S SO OFF THE MARK WHAT IT
COSTS I DON'T KNOW WHY IT'S
THERE.
>> THE PERCENTAGE MODULAR IN OUR
INSTITUTE NOW IS QUITE LOW.
IT'S SELF-SELECTED OUT.
>> I THINK THE LAST NUMBERS WE
HAD IN 2020 IS 80% MODULAR IS
AROUND 10%.
OKAY.
THE OTHER THING WE PAINFULLY
HAVE DONE IS TO INCREASE OUR
ADMINISTRATIVE CUTS.
WE DID THAT LAST YEAR.
TALKED ABOUT THIS BEFORE.
IN THE TIME OF THE DOUBLING
PEOPLE WERE PUTTING IN REAL
EXPENSIVE GRANTS AND NIH STARTED
TO PUT IN ADMIN CUTS TO REDUCE
THE COST OF THE GRANTS AND THAT
WAS IN A GOOD TIME.
ANYWAY, NINDS GOT TO 17.5% IN
THE RO1 MODULAR BACK IN THOSE
DAYS AND WERE NEVER ABLE TO GET
RID OF IT AND ADDED 3% ON LAST
YEAR BECAUSE OF THE CRISIS MODE
AND THEY WERE KWAPD SO -- CAPPED
YOU WILL END UP AROUND 400.
THIS IS THE DATA ON THE
FELLOWSHIPS.
THE F GRANTS DROPPED BY NUMBER
OF FIVE.
A VERY SMALL PERCENT.
THE CAREER DEVELOPMENT AWARDS,
THE Ks DROPPED BY 11.
AGAIN, A SMALL PERCENT AND THE
T32s DROPPED TO BY TWO AT 79.
EVERYTHING TOOK A LITTLE BIT OF
A HAIRCUT BUT IT'S MORE
PRESERVED THAN ANY OTHER PART.
OKAY.
THAT'S THE NEWS.
SOMETHING TO BRIGHTEN OUR
COUNCIL TO SHOW THE REPORT OUT
OF THE NEW ENGLAND JOURNAL OF
MEDICINE NOT JUST THE ONE THING
BUT THE BRAIN INITIATIVE HAS
JUST ABOUT ALL THESE TOOLS WE
RECORD FROM THE NERVOUS SYSTEM
THAT CAN MODULATE THE NERVOUS
SYSTEM AND WORKING HARD TO MAP
THE NERVOUS SYSTEM.
THESE ARE GOING TO BE POWERFUL
IN TERMS OF TREAT BEING
DISEASES.
NOW, THIS PAPER KIND OF
ILLUSTRATES THE ISSUE OF WHEN I
STARTED IN TRAINING PEOPLE WOULD
STICK AN ELECTRODE IN ONE NEURON
IN A MONKEY AND SHOW A VISUAL
TASK AND RECORD AND PUT ANOTHER
ONE IN AND NEURON BY NEURON NOW
PEOPLE CAN RECORD FROM
MILLINEURONS WITH OPTICAL
TECHNIQUES.
THIS IN HUMANS IS RECORDING FROM
254 NEURONS IN ALS.
A COMPLETELY PARALYZED PERSON
BUT THEY'RE ABILITY TO ENGAGE
THEIR BROKERS AREA TO SPEAK IS
PRESERVED.
AND WHAT THEY DO IS PUT THESE --
THEY PUT FOUR OF THESE
ELECTRODES ARRAYS INTO THE LEFT
SIDE OF THIS PERSON'S BRAIN IN
THE REGIONS YOU SEE ON THE BRAIN
PICTURED BELOW THE MOTOR BROKERS
AREA AND SEE IN THE LITTLE BOX
PLOE THE ELECTRODES IS THE
FIRING AND THE PROBLEM IS NOT
BEING ABLE TO RECORD BUT WHAT'S
IT MEAN.
YOU KNOW FROM WHAT TO THE
PERSON'S TRYING TO DO THERE'S A
CODE IN THERE AND THEY'RE TRYING
TO IDENTIFY PHONEMES FOR FIRING
PATTERNS AND INTO A LARGE
LANGUAGE NETWORK AND TAKE
PHONEMES AND IDENTIFY THE NEXT
WORD LIKE CLASS GPT DOES AND
MAKES A HUGE ABILITY FOR THIS
PERSON TO COMMUNICATE ALMOST AT
IN A CONVERSATIONAL MANNER.
CAN YOU GUYS GET THE VIDEO TO
GO?
THIS IS BRAIN GATE, U.C. DAVIS.
YOU SEE ON THE SCREEN HE'S
THINKING WHAT HE WANTS TO SAY
AND IT'S SHOWING UP IS ON THE
SCREEN.
HE PUSHES A GREEN BUTTON.
CAN YOU TURN UP THE VOLUME,
ANYBODY?
THAT IS HIS VOICE RECORDED
BEFORE HE LOST ABILITY TO SPEAK.
HE WAS ABLE TO USE THE ENTIRE
ENGLISH VOCABULARY WITHIN A
COUPLE DAYS.
>> WAS THIS ADVANCE MADE THROUGH
BRAIN INITIATIVE FUNDS?
>> I THINK THE GRANT IS FROM
NIBCB.
I THINK IT REQUIRES A STANDING
OVATION.
>> THIS IS FROM THE PERSPECTIVE
OF PEOPLE WHO MAYBE AREN'T AS
LUCKY BUT THIS IS THE SAME
TREATMENT.
>> I HOPE WE'RE CLOSE TO THE
TIME WHERE EVERYBODY HAS THE
OPTION I DO.
>> PRETTY AMAZING STUFF.
>> HE'S ABLE TO WORK NOW.
HE WAS COMPLETELY PARALYZED.
THIS IS CRITICAL.
THE NEUROSCIENCE 50 YEARS AGO TO
NOW IT'S UNBELIEVABLE TO THINK
WHAT THINGS WILL LOOK LIKE 50
YEARS FROM NOW.
THIS IS JUST BEING ANNOUNCED
LIKE TODAY, I THINK IT IS.
IT'S A PROJECT WE'RE WORKING
WITH THE NATIONAL EYE INSTITUTE
WHO IS LEADING THIS.
TRYING TO DEVELOP WHAT'S CALLED
AN S INDEX OR DATA SHARING
INDEX.
AS YOU KNOW, NIH MADE DATA
SHARING MANDATORY FOR ALL THE
GRANTS.
AND THIS IDEA IS TO REWARD
PEOPLE WHEN THE DATA THEY SHARE
SHOWS VALUE TO THE SCIENTIFIC
COMMUNITY.
THE CHALLENGE IS FOR FOLKS TO
WORK OUT A WAY IN WHICH THAT
COULD BE DONE FAIRLY AND THAT'S
EVALUATING THE VALUE OF WHAT HAS
BEEN POSTED FOR THE SCIENCE
COMMUNITY IN THE SHARING AND
THERE'LL BE A PHASE I WHERE
PEOPLE SUBMIT IDEAS AND PICK
SOME FINALISTS AND THEY GET SOME
MONEY AND THEN GET TO COMPETE
FOR THE PHASE II WHICH IS WHERE
THE $1 MILLION AT THE END AND WE
HAVE AN H INDEX FOR PEOPLE
LOOKING AT PUBLICATIONS.
THIS WOULD BE FOR LOOKING AT
DATA WHICH SOME DAY WILL
SUPPLANT THE H INDEX.
THE REAL VALUE OF THE SCIENCE IS
IN THE DATA.
SOMEONE TOLD ME ONCE THE DATA IS
THE CORE AND EVERYTHING ELSE IS
AN ADVERTISEMENT.
WE'LL LEAR LATER FROM AMBER AND
FRANK FROM THE ADRD RESEARCH
GOING ON.
WE'RE GUIDED IN THIS RESEARCH BY
A SUMMIT WE HOLD EVERY THREE
YEARS.
THE NEXT ONE WILL BE IN 2025.
AND THERE'S A WORKING GROUP OF
COUNCIL ON THE EXPOSOME AND
DEVELOPED PRIORITIES FOR NINDS
AND TO CUT TO THE QUICK WHEN YOU
STUDY DISEASE AND LOOK AT
GENETIC DRIVERS, WE'VE BEEN
SUCCESSFUL AT UNDERSTANDING THEM
BUT WE'RE GENERALLY FINDING
WE'RE HITTING THE CEILING AND WE
MIGHT EXPLAIN 17% OR 20%.
THAT MEANS 80% IS COMPLETELY IN
THE DARK AND WE BLAME IT ON THE
ENVIRONMENT WHICH IS EVERYTHING
ELSE NOT GENETIC SO IT'S A BIG
SPACE.
THERE'S IMPORTANT THINGS OUT
THERE AND THE QUESTION IS DO WE
HAVE THE TOOLS TO BE ABLE TO
NAIL SOME OF THESE DOWN BECAUSE
IF YOU CAN YOU CAN POTENTIALLY
PREVENT THINGS AND MODIFY RISKS.
THAT WOULD BE FANTASTIC.
THIS GROUP YOU SEE HERE HAS BEEN
WORKING OVER THE LAST YEAR AND
LOOK FORWARD TO THEIR REPORT.
THE HEAL INITIATIVE MOVED FROM
THE OFFICE OF THE DIRECTOR NOW
RUN BY NIDA AND NINDS AND THEY
RUN THE PAIN SPACE WITH 14 OTHER
DIRECTORS AND WELL RESOURCED
OVER $3 BILLION GOING TO THE
HEAL SINCE IT'S BEGUN.
THERE'S BEEN 40 FDA APPROVALS
FOR INVESTIGATIONAL DEVICES AND
CLINICAL TRIALS ARE ONGOING AND
THERE WAS NEVER A STRATEGIC
PLAN.
IT WAS BORN WITH DR. COLLINS'
INPUT FROM THE BEGINNING TO
RESPOND TO A CRISIS AND NOW WE
FEEL ON THE PAIN SIDE IT'S
IMPORTANT TO DEVELOP A STRATEGIC
PLAN TO GUIDE THE HEAL
INITIATIVE FORWARD.
WE'RE PLEASED ROB GERREAU AGREED
TO HELP DIRECTOR THE FUND AND
HAVE AREAS WE THINK THE GROUP
NEEDS TO TAKE DEEP DIVES INTO
AND HAVE CHOSEN EXPERT FOLKS TO
LEAD THE SUBCOMMITTEES.
THEY'LL BE WORKING OVER THE NEXT
YE
YEAR.
THERE WAS A SERIES OF RESEARCH
PRIORITIES FROM THE HEADACHE
RESEARCH I THOUGHT WAS
COMPREHENSIVE AND POTENTIALLY
RELATED GIVEN HEADACHE AND PAIN
ARE PAIN DISORDERS AND ACTUALLY
RIPE FOR MOVING FORWARD IN THE
HEAL INITIATIVE AND MICHAEL
OCHINSKY TOOK PART IN THOSE FOR
THE HEADACHE SOCIETIES.
YOU'VE SEEN CLINICAL TRIALS IN
SUB-SAHARAN AFRICA.
WE HAVE PUT INVESTMENTS INTO
GLOBAL HEALTH FOR ONE OF TWO
REASONS.
ONE IS TO STUDY DISORDERS MORE
EASILY STUDIED IN LOW AND MIDDLE
INCOME COUNTRIES LIKE MALARIA
AND T.B., MENINGITIS.
PEOPLE THAT AFFECT PEOPLE IN THE
U.S. BUT RAMPANT IN LOW AND
MIDDLE INCOME COUNTRIES.
IN RESEARCH WE'LL HAVE A BIG
IMPACT IN U.S. HEALTH AND
IMPROVES THE HEALTH OF THE LOCAL
AREA AND THE SECOND REASON
SOMETIMES WE HAVE THE
OPPORTUNITY TO MAKE A MODEST
INVESTMENT WE THINK CAN REALLY
IMPROVE NEUROLOGICAL HEALTH IN
DRAMATIC WAYS IN SOME OF THE LOW
AND MIDDLE INCOME COUNTRIES.
WHAT RICHARD BENSON WITH OTHERS
IS HOPING TO DEVELOP STRATEGIC
PLANNING AND THEY'LL LOOK FOR
VOLUNTEERS FOR THE COUNCIL
INTERESTED WORKING ON THE
WORKING GROUP DEVELOPING A ROAD
MAP FOR GLOBAL HEALTH RESEARCH
AND THESE ARE THE KINDS OF
THINGS THAT WILL TAKE A YEAR AND
2026 WILL TAKE A YEAR AND
HOPEFULLY WE'LL HAVE FUNDING TO
MOVE THINGS FORWARD WITH THE
PLANS COMING OUT IN TIME.
IT'S GOOD TIME BEING.
-- TIMING.
IN TERMS OF HEALTH EQUITY IN THE
U.S.
THEY DID EXTENSIVE STRATEGIC
PLANNING PUBLISHED AN ISSUE IN
NEURO O
NEUROLOGY WHICH HAD GRANTS AND
YOU SEE COMBAFT BUILDING FOR
DOING OUR RESEARCH TO REDUCE
HEALTH DISPARITIES IN
NEUROLOGICAL DISORDERS.
6:
THE OTHER REPORTS WAS ON THE
NEUROSCIENCE WORKING GROUP AND
THERE'S A WORKSHOP BEING SET UP
FOR MARCH 11 TO LOOK AT SPATIAL
CHARACTERIZATION OF MOLECULES IN
VIVO AND MEASURING CELLULAR
ACTIVITY IN VIVO ALL HIGH POINTS
FROM THE WORK GROUP'S REPORT.
AND AGAIN, HOPING TO MOVE THINGS
FORW
FORWARD BUT MOVING FORWARD IN
THE BUDGET ISSUES BEFORE WE CAN
MOVE FORWARD IN THE SPACE.
THERE WAS A NON-PROFIT FORUM IN
THE SUMMER WHICH WAS ONCE AGAIN
BETTER THAN EVER.
EVERY YEAR I SAY THAT AND IT'S
TRUE.
THIS WAS GREAT.
THEY HAD TOPIC AND ONE ON
ARTIFICIAL INTELLIGENCE GROUPS
REPRESENTING DATA AND THEIR DATA
IS BEING TAKEN AND USED FOR
DIFFERENT PURPOSES.
AND THE NON-PROFITS ARE REALLY
INTERESTED IN THIS SPACE RIGHT
NOW AND THEN WE GOT A GREAT KEY
NOTE DISCUSSION FROM MINDY URLAB
WHO HAS BEEN COURAGEOUS IN
MOVING FORWARD HER NONPROFIT AND
LAD -- HAD A GREAT STORY TO
TELL.
THERE USED EPILEPSY AND THEN
EPILEPSIES AND NOW DUE TO THE
FACT SO MANY COMBRCHK
GENETIC MUTATIONS HAVE BEEN
DISCOVERED AND THERE'S A HUNDRED
DISEASE GROUPS RELATED TO A
PARTICULAR GENETIC CAUSE OF
EPILEPSY AND THESE GENOMIC
THERAPIES HAVE THE POTENTIAL TO
MAKE A DIFFERENCE AND THAT'S
WHAT THE MEETING WAS ABOUT.
IT DOVETAILS WITH WHAT WE'LL BE
HEARING FROM THEODORE AND ALSO
TO THE WORK BEING DONE IN THE
SOMATIC CELL GENE EDITING
PROGRAM AND UNDIAGNOSED DISEASE
NETWORK AND OUR OWN NETWORK TO
FUND GENE-BASED THERAPIES AND
NEUROLOGICAL DISORDERS CALLED
URGENT AND PART OF THE
ACCELERATING MEDICINE FOLKS TO
BUILD A PLATFORM USING A B VIRUS
THAT COULD BE MORE PLUG AND
PLAY.
ONCE IT'S ACCEPTED BY THE FDA
AND YOU HAVE SYMPTOMS IN YOU CAN
PUT ONE GENE THERAPY TO THE SAME
VECTOR AND KEEP REPEATING THAT
BUT NOT STARTING FROM GROUND
ZERO EVERY TIME TO TRY TO GET
SCALE UP.
>> GOOD LUCK.
>> WITH THAT WE'LL HEAR FYODOR
THE DIRECTOR OF TECHNOLOGY AND
TRANSLATION AT THE INNOVATIVE
GENOMICS INSTITUTE CALIFORNIA
BERKELEY AND PUT OUT A PAPER HOW
TO SCALE UP THE GENETIC
MEDICINES CALLED THE ROAD MAP OF
GENETIC MEDICINES IN NATURE AND
THE TAKE HOME FOR ME AS COMPARED
TO DRUG DEVELOPMENT WHERE ALL
THE IP IS ON THE MOLECULE AND
IT'S FORTIFIED THE GENES AND
MUTATIONS ARE PUBLIC KNOWLEDGE
AND DEVELOPING A GENE THERAPY
WITH AN ASO OR REPLACING A
MISSING GENE IS OPEN FOR
ACADEMICS TO DO.
THEY DON'T HAVE TO GO THROUGH
ALL THE MEDICINAL CHEMISTRY AND
THE SCREENING THE DRUG COMPANIES
DO FOR SMALL MOLECULES.
THE QUESTION IS HOW DO YOU SCALE
UP AND PARTICULARLY HOW DO YOU
DEAL WITH THE ISSUE OF THE
PRICING OF THE AGENTS AS THEY
COME OUT.
SOME THERAPIES NOW ARE $3
MILLION A SHOT.
I DON'T THINK THE MANUFACTURING
IS CLOSE.
ASOs RELATIVELY INEXPENSIVE.
THE COMPANIES HAVE TO COVER THE
COST OF THEIR DEVELOPMENT.
IF NIH COVERS THE COST OF
DEVELOPMENT COULD WE DEVELOP
THESE FOR THE ULTRA RARE
DISORDERS WHERE IS NOT THAT
INTERESTED AND PROVIDE THEM AT
COST, FOR INSTANCE.
THAT'S THE THING THAT ALLOWS
THINGS TO EXPAND RAPIDLY.
INTERESTING TO HERE WHAT FYODOR
SAYS.
THAT'S THE END AND OUR STORY AND
WE'RE STICKING TO IT.
>> THANK YOU FOR THE OVERVIEW
AND FOR WHAT YOU DO.
TWO COMMENTS.
ONE, I APPRECIATE YOUR HOLDING
THE LINE ON TRAINING GRANTS.
IT'S COST EFFECTIVE AND
INSURANCE FOR THE FUTURE.
WE NEED TO MAKE SURE WE DO THAT.
SECOND COMING BACK TO YOUR
EARLIER POINT, DR. BERTAGNOLLI
IS INTERESTED IN TRIALS IN THE
COMMUNITY AND IN PREVENTATIVE
MEDICINE.
I SUPPORT THAT.
TAKING ALZHEIMER'S DISEASE 50%
OF THE RISK IS MODIFIABLE RISK
FACTORS PEOPLE DON'T KNOW ABOUT
TO PREVENT.
WITH OUR LIMITED PIE AND ALL THE
GREAT WORK WE DO IN FUNDAMENTAL
NEUROSCIENCE AND PRECLINICAL AND
HOW DO WE MAKE ROOM FOR
ADDITIONAL WORK TO HAVE
MECHANISMS TO GET IN THE SPACE
OF MODIFIABLE RISK FACTORS TO
THE PUBLIC.
>> I THINK THE BIGGEST PUBLIC
HEALTH IMPACT WILL BE IN
PREVENTION.
MY SENSE IS NO ONE AS A BEEN
CLEVER ENOUGH TO FIGURE HOW TO
DO BEHAVIOR CHANGE.
WE HAD A WORKSHOP ON BEHAVIORAL
CHANGE AND HAD BEHAVIORAL
ECONOMICS FOLKS COME AND THERE
ARE WAYS IN WHICH YOU CAN MODIFY
BEHAVIOR BUT THE LEVERS EXIST IN
THE COMPANIES.
IF YOU'RE AN EMPLOYEE OF A
COMPANY AND THE COMPANY HAS AN
OPT-OUT CLAUSE.
EVERYBODY USES THE GYM ONCE A
DAY, YOU HAVE TO OPT-OUT, THOSE
KINDS OF THINGS CAN WORK BUT IT
WAS HARD TO SEE HOW AND THE RISK
FACTORS WE WENT AFTER IN THE
SPRING PROGRAM WHICH WAS BLOOD
PRESSURE LOWERING LOOKING AT
COGNITIVE IMPAIRMENT OF DEMENTIA
WE HAD TO CLOSE IT DOWN BECAUSE
IT REDUCED MORTALITY.
IT WAS UNETHICAL TO GO FURTHER.
SO THERE WERE SO MANY REASONS
WHY PEOPLE SHOULD BE AGGRESSIVE
WITH THEIR BLOOD PRESSURE BUT
DOING A CLINICAL TRIAL IS NOT
FEASIBLE FOR THAT REASON THOUGH
WE GOT A PRETTY GOOD SIGNAL
THOSE FOLKS WHO HAD THE
AGGRESSIVE BLOOD PRESSURE HAD
LESS COGNITIVE DEMENTIA.
THE ISSUE IS BEHAVIOR CHANGE.
>> IT BRINGS A GREAT EXAMPLE
BECAUSE I THINK MANY CLINICIANS
WILL HIGHLIGHT THAT STUDY WHEN
THEY TALK TO PATIENTS SAYING WE
KNOW THIS MAKES A DIFFERENCE.
MAYBE PART OF WHAT WE'RE TALKING
ABOUT IS MORE STUDIES THAT SHOW
PREVENTIVE APPROACHES MAKE A
DIFFERENCE.
IT'S A GOOD EXAMPLE.
>> SOME OF THE CLEVER IDEAS CAN
MAKE A BIG DIFFERENCE.
>> THANK YOU FOR THAT OVERVIEW.
VERY INFORMATIVE.
NOT TO DRAG US BACK DOWN TO THE
SOBERING ISSUES OF BUDGETS BUT A
QUESTION ABOUT THE APPROACH IN
THINKING ABOUT THE
ADMINISTRATIVE CUTS WHICH HAVE
BECOME SO NECESSARY, I DO WONDER
IF THEY LEAD TO ANTICIPATORY
INFLATION.
PEOPLE ARE THINKING IF THEY'LL
GET FUNDED WILL GET A CUT.
DOES IT MAKE A SENSE OF THE
MODULAR BUDGET OF FINDING A
MODULAR BUDGET FORMAT WITH A
THRESHOLD AND HAVING PEOPLE TO
STICK OR GO FOR THAT WITH THE
MOTION THEY'RE GOING TO FIND THE
MOST EFFICIENT COST EFFECTIVE
WAY OF DOING THINGS AND IF THEY
CAN MEET THAT THRESHOLD THEN
THEY WON'T BE SUBJECT TO THE
CUTS.
I WONDER IF IT BECOMES THE
VICIOUS CYCLE OF THE MORE CUTS
YOU'LL GET THE MORE YOU'LL SEE
INFLATED BUDGETS TO COUNTER THAT
OR IF YOU'VE SEEN EVIDENCE OF
THAT.
>> THE APEOPLE --
>> THE PEOPLE WHO APPLY FOR
GRANTS ARE CLEVER.
YOU HAD TO GET APPROVAL FOR A
GRANT OVER $500,000.
AND I FORGET THE NUMBER, BRIAN
KNOWS, BUT IT'S SOMEWHERE 50% TO
60% OF THE GRANTS ARE R90.
PEOPLE ARE COMING IN FOR THE
MAX.
WE THOUGHT ABOUT WITH THE SPLIT
PAY LINE WE THOUGHT ABOUT MAKING
THAT AT A LEVEL THAT WOULD BE
SOMETHING THAT'S REASONABLE BUT
THEN THE QUESTION IS WHAT IS
THAT NUMBER.
SO IF YOU ASK AROUND AND THIS IS
ANECDOTAL TO A GRANT YOU NEED
AROUND $350K.
THAT'S THE GUESS.
CLEARLY 250K FOR THE MODULAR IS
TOO LOW AND 350 AND 275 WITH THE
20% CUT WE'RE AT 400 SO WE'RE
PRETTY MUCH THERE.
THEN WE HAVE THE PRECEDENT OF
NIA WITH A SIMILAR SPLIT SO
YOU'RE RIGHT THAT'S WHAT'S
LAPENING.
BECAUSE OF THE PAY LINE WE DO
CONTROL IT NOW.
>> WALTER WOULD IT BE FAIR TO
SAY THE CHANGE IN THE MODULAR
HAS BEEN BROUGHT UP WITH I.C.
DIRECTORS AND FOR SOME REASON IT
JUST CAN'T GET THERE.
YOUR POINT IS NOT LOST.
>> IT'S IMPORTANT TO SAY ARE THE
BUDGETS INFLATED WHEN THEY'RE
475, 480?
WHEN YOU LOOK AT THE COST OF
INFLATION THEY'RE THE EXACT SAME
AS 30 YEARS AGO SO WE'RE SAYING
THEY'RE INFLATED AND THERE'S
MENTAL PADDING IT BUT WITH
POSTDOC SALARIES GOING UP I
THINK THE IDEA THAT WE'RE SAYING
WE'RE OVER INFLATING OUR BUDGETS
IS INCORRECT WHEN YOU LOOK AT
THE PRICE OF REAGENTS MINUS
STAFF WE HAVEN'T INFLATED THE
BUDGETS AT ALL.
THEY'RE EXACTLY FLAT.
>> TAKE INTO ACCOUNT THE MPI
APPLICANT THE ONES IN THE HIGHER
ECHELON ARE THOSE.
>> BUT THE COST OF DOING
RESEARCH IS DOUBLED.
>> YOU HAVE TO LOOK AT
INFLATION.
I DON'T KNOW WHERE YOU CAN CUT
BUT I'M HAPPY THE TRAINING
PROGRAMS SHOULD ALWAYS BE
PRESERVED AS TO THE EARLY STAGE
INVESTIGATOR.
AND JUST A JUMP IS CONCERNING
FOR OUR PIPELINE.
I THINK THOSE ARE THE THINGS WE
REALLY NEED TO PROTECT AND GET
TO BE CREATIVE WHETHER WE
PARTNER MORE WITH INDUSTRY FOR
THE EXPENSIVE CLINICAL TRIALS OR
USING DEVICES WE'LL HAVE TO GET
CREATIVE WITH FUNDING BECAUSE
THE COST OF RESEARCH IS NOT
GOING TO GO DOWN.
>> AS SOMEONE FOOLISH ENOUGH TO
STILL PRESENT A MODULAR BUDGET I
WONDER IF WE CAN RAISE THAT 16
PERCENTILE BY STOPPING FUNDING
THOSE GETTING OVER $1.5 MILLION
A YEAR.
AS AN INVESTIGATOR I FEEL
BETTER.
I'M INVOKING THE WRATH OF THOSE
GETTING A LOT OF FUNDING I'M
SURE BUT MAYOR THERE'S SOMETHING
TO BE SAID ABOUT NOT CUTTING
PERCENTILE, 16% IS CRAZY LOW.
MAYBE WE SHOULD THINK OF
REDISTRIBUTING THE FUNDS JUST A
LITTLE MORE THAN WE'RE DOING
NOW.
>> I FULLY AGREE WITH THAT
SENTIMENT AND WHEN I HAVE THAT
THOUGHT I HAVE THE COUNTER
THOUGHT AS WE'RE TRYING TO
CREATE ALL THESE K LEVEL PEOPLE
IN THE PICTURE.
THEY HAVE TO HAVE MENTORS AND
THEY HAVE LARGE LABS.
COULD WE INCORRECTLY FUND THE
LABS VIA THE K BY PROVIDING SOME
SALARY OR PROVIDING MORE THAN
THE MINIMAL SUPPLIES THAT COULD
ACTUALLY REALLY COVER THE WORK
OF THAT.
IT'S JUST THINKING ABOUT IT
DIFFERENTLY.
I WORRY IF WE SHRINK OUR LARGER
LABS WE MAY LOSE THE TRAINING.
>> I LOVE THAT.
GIVING MONEY THROUGH THE LARGER
LABS THROUGH THE TRAINING AWARDS
LINKED TO THE CAPACITY AS WELL.
YOU DON'T WANT TO LOSE THAT AND
IT'S A DEBATE INSIDE MY OWN HEAD
AND WONDER IF WE MIGHT ADDRESS
IT.
>> IN TERMS OF THE ESIs THEY'RE
EARLY STAGE.
THEY'RE OF WE'RE GOING TO SEEK
YOUR APPROVAL TO GO WHERE WE
BELIEVE THE PAY LINE WE HOPE
WILL END UP.
SO WE STILL WOULD SEEK THE
AUTHORITY TO GO BEYOND THE 60
MARK.
>> I WASN'T TOO CLEAR WITH THE
TRAINING.
I NOW WANT TO MAINTAIN THAT AS
MUCH AS POSSIBLE.
HOW MUCH OF THE F GRANTS AND SO
WILL THEY HAVE?
I'M WONDERING WHETHER THERE'S
SOME KIND OF INCENTIVE FOR
INVESTIGATORS TO BE PUSHING TO
OFFSET THE COST.
I THINK ONE OF THE MOST
EXPENSIVE PART OF THE BUDGET FOR
AN INVESTIGATOR IS THE SALARIES.
PUSHING AN INCENTIVE TO APPLY
FOR MORE THE F AWARDS FOR AT
LEAST FOR THE STUDENTS.
NOT SURE THAT SHIFT THE PROBLEM
ELSEWHERE BUT I CONFESS I'M ALSO
A MODULAR BUDGET GUY BUT IT'S
GETTING HARD AND WE GO FOR THE F
GRANTS BUT THOSE ARE JUST AS
BRUTAL GET AS AN RO1.
SOME INCENTIVE MAY BE HELPFUL.
>> I WANT TO THANK TISH WIGAN
AND THERE'S A SERIES OF FORCES
COMING UP THAT WEREN'T THERE 20
YEARS AGO.
NEGOTIATING THEM AND THEIR
KANSAS TRICKY BUT THERE'S
PROBABLY THINGS WE CAN DO
DIFFERENTLY TO GET US TO THE
GOAL WE WANT.
IN TERMS OF POSTDOCS, THE Fs
ONLY ACCOUNT FOR 3% OF ALL THE
POSTDOCS ON RO1s.
THEY'RE GOAL WAS TO PUSH THE Fs
TOWARDS TO THE POSTDOC F YOU
HAVE TO APPLY WITHIN A SHORT
TIME AFTER JOINING THE LAB WITH
THE IDEA BEING FUNDING SOMEBODY
WHO IS GOING TO WORK THEIR OWN
SPACE IN SOMEONE'S LAB AND THEN
HAVE SOMETHING TO GO OUT WITH.
SIMILARLY THE Ph.D. PROGRAMS THE
JOINT T32s FOR THE BLUEPRINT FOR
NEUROSCIENCE AND THERE I THINK
WE HAVE THE ABILITY TO -- WE
MADE SOME LITTLE TWEAKS BUT I
THINK MORE ABILITY THERE.
I THINK WE HAVE TO THINK ABOUT
TRAINING BIG PICTURE WISE IN THE
NEXT YEAR OR SO.
>> I'D MAKE MORE COMMENTS ABOUT
THE TRAINING ASPECT.
IT'S ONE THING TO SUPPORT THE
TRAINEES BUT WE'RE ALSO LOSING A
FAIR NUMBER OF TRAINEES WHEN
THEY ARE SEE WHAT HAPPENS
FURTHER DOWN THE ROAD AT THE
FUNDING LEVELS AND DECIDE THIS
IS NOT AN AVENUE FOR THE MOST
INTELLIGENT PEOPLE TO MOVE
FORWARD AND BALANCING THAT WHAT
WE'RE DOING TO THE MID CAREER
PROSPECTS OF THESE INDIVIDUALS
WE'RE SPENDING SO MUCH TIME
TRAINING BECOMES ESSENTIAL.
>> THIS IS PROBABLY THE WRONG
TIME TO BRING THIS UP WITH TO
THE BUDGETS BEING SO POOR BUT
SEVERAL OF US ON THE WALK OVER
HAD A DISCUSSION HOW EXPENSIVE
IT IS TO TRAIN INVESTIGATORS.
AND I'VE ESTIMATED IT'S ABOUT
$75,000 A YEAR PER K AWARDEE AND
THEN THE COST THAT GOES BEYOND
THAT IN TERMS OF WHEN THEY GET
THEIR FIRST RO1 TO PROTECT THEIR
TIME FOR THE SECOND.
LOOKING AT THE NUMBERS WE
HAVEN'T CHANGED THE BAR IN TERMS
OF THE NUMBERS OF NEW
INVESTIGATORS.
I THINK GOING FORWARD WHEN
BUDGETS ARE BETTER WE MIGHT
THINK ABOUT HOW TO SUPPLEMENT
THE COSTS ON THE NEUROLOGY
DEPARTMENTS THAT DON'T BRING IN
REVENUE.
I THINK THAT'S GOING TO BE
REALLY IMPORTANT.
WE HAVE 19K AWARDEES AND I LOSE
SLEEP ON HOW MUCH THAT WILL COST
US OVER THE NEXT FIVE, 10 YEARS.
>> PERHAPS THE LAST QUESTION.
>> I'M CURIOUS WHEN I THINK
ABOUT WHEN I STARTED MY
INDEPENDENT CAREER OVER TWO
DECADES AGO PUSHING THREE, THIS
WAS THE ONLY GAME IN TOWN.
200 NEUROSCIENCE RESEARCHERS AND
IN THAT TIME SO MANY HAS CHANGED
THERE'S OTHER PLAYERS THAT ARE
CHANGING THE ECO SYSTEM.
NOT ONLY SHOULD WE HAVE BETTER
BUDGETS BUT KHAN ZUCKERBERG AND
SIMON, TO NAME THREE EXTREMELY
GOOD PHILANTHROPISTS FOR SCIENCE
BUT ALSO THE DEPARTMENT OF
DEFENSE JUST LOOKING AT THE
PAPER ADVERTISED WITH GREAT
SUCCESS WAS FUNDED BY DOD.
AT LEAST THAT'S WHAT THEY'RE
GIVING CREDIT FOR IN THE FRONT
LINE OF THE PAPER AND YOU
COULDN'T HAVE AFFORDED IT IF
THEY SAID DON'T PAY MORE THAN
$1.5 MILLION.
HAS ANYONE LOOKED AT TO THE
WHOLE ECO SYSTEM TO LOOK AT THE
BUDGETS AND IT MIGHT TEMP PERRER
-- TEMPER LOOKING AT THE WHOLE
PACKAGE RIGHT NOW AND I'M
GATHERING IT HASN'T BEEN DONE.
>> I HAVEN'T SEEN ANYBODY DO
THAT YET.
I THINK NIH IN A SENSE IS YOUR
BREAD AND BUTTER AND THE OTHERS
ARE MORE NICHE AREAS.
YOU HAVE TO HAVE THAT FOUNDATION
NINDS OFFERS.
MOST THE PEOPLE YOU MENTION THE
FOLKS MENTIONED FUND WERE FUNDED
BY NINDS FIRST AND THE INSTITUTE
SO RIGHT --
>> ZUCKERBERG HAS A YOUNG
INVESTIGATOR AWARD.
NOT THE SAME EXTENT AND I FEEL
LIKE THERE'S ROOTS IN ANALYSIS
SAYING WHAT IS THE WHOLE ECO
SYSTEM IN TERMS OF NEUROSYSTEM
FUNDING.
YOU CAN'T LOOPING -- LOOK AT
WHAT THE SIMONS FOUNDATION WILL
DO BUT FOR THE LONG HAUL.
>> AND NEUROSCIENCE FUNDING
INCREASED AT NIH MORE GOES TO
NEUROSCIENCE THAN ANY OTHER
AREA.
WE'RE CRYING BUT WE HAVE HALF A
BILLION A YEAR.
THINGS GO UP AND DOWN AT THE NIH
LEVEL AND WE HAVE TO TRY AND
RIDE THAT WAVE AND NOT DO
PERMANENT DAMAGE AND WE DO WANT
TO PARTNER OR COORDINATE,
PARTNER, WORK WITH ALL THE OTHER
AGENCIES WHO ARE INVESTED IN
THIS SPACE THEIR DOLLARS ARE
ALMOST AS GOOD AS OUR DOLLARS.
TRUTH IS IT'S JUST GETTING THE
WORK DONE.
WE'RE DEFINITELY OPEN TO
ANYTHING WE CAN DO FOR SURE.
TO ADVANCE THEIR VISION.
>> SO WITH THAT, THANK YOU,
WALTER.
THANKS FOR ALL THE QUESTIONS AND
DISCUSSION.
I DO THINK WE NEED TO MOVE LONG
TO OUR ADRD UPDATE.
WE'LL PASS IT ON TO AMBER
MCCARTNEY AND FRANK SHOEMAKER
FROM THE DIVISION OF
NEUROSCIENCE TO GIVE US A BRIEF
UPDATE.
IT'S BETWEEN YOU TWO AND LUNCH.
>> HI, EVERYBODY.
I'M THE DIRECTOR FOR TEMPORO
FRONTAL DEMENTIA AND I'LL TALK
ABOUT OUR PROGRAM.
IT'S PART OF A LARGER TEAM
EFFORT WITH THE NATIONAL
INSTITUTE ON AGING IN WHICH WE
SUPPORT RESEARCH ON ALZHEIMER'S
DISEASE AND THEY'RE THE LEAD ON
AD AND WE'RE THE LEAD ON ADRD.
NORMALLY ROD WILL GIVE THE
PRESENTATION BUT HE'S ON LEAVE,
A FANTASTIC PROGRAM MANAGER AND
AMBER AND I WILL GIVE THE UPDATE
IN HIS PLACE.
I WANT TO THANK A FANTASTIC TEAM
THAT SUPPORTS OUR ADRD PROGRAM.
GREAT EXPERTISE ACROSS THE
BOARD, POLICY, GRANTS MANAGE,
FINANCE.
I WANT TO HIGHLIGHT PEOPLE WHO
PUT THE PRESENTATION TOGETHER.
AMBER AND ROBIN AND IN THE
MIDDLE COLUMN I WANT TO THANK
OUR SCIENTIFIC REVIEW BRANCH.
THEY REVIEW THE APPLICATIONS AND
WE COULDN'T DO THIS WITHOUT THEM
AND ON THE FAR RIGHT PROGRAM
STAFF AND LET ME INTRODUCE A NEW
PROGRAM DIRECTOR WE HAVE XILING
YIN FOR VASCULAR CONTRIBUTION
FOR COGNITIVE IMPAIRMENT AND
DEMENTIA AND JUST STARTED AND
WE'RE EXCITED TO HAVE HER IN
THIS ROLE.
NO SURPRISE, ADRD IS A NATIONAL
PRIORITY.
THE LOSS OF COGNITIVE DECLINE IS
DEVASTATING TO INDIVIDUALS AND
FAMILIES.
IT'S A MAJOR HEALTH ISSUE AND
TAKES UP A LOT OF OUR PUBLIC
HEALTH RESOURCES.
A SHOUT OUT TO OUR
COMMUNICATIONS TEAM.
THEY PUT TOGETHER A GREAT
YOUTUBE VIDEO THAT EXPRESSES THE
CHALLENGE OF ADRD THAT EXPRESSES
THE CHALLENGE BETTER THAN I CAN.
MORE THAN A DECADE AGO CONGRESS
DEVELOPED THE ALZHEIMER'S
PROJECT ACT AND CALLED FOR A
NATIONAL PLAN, A UNIFIED PLAN TO
ADDRESS ALZHEIMER'S DISEASE AND
2012 THE FIRST NATIONAL PLAN WAS
CREATED.
EVERY YEAR SINCE 2012 THIS IS
UPDATED WITH THE STATE OF THE
SCIENCE AND UNDERSTAND THE
DIFFICULTY OF DIAGNOSIS AND
PATHOLOGY AND IT CAN BE HARD TO
DRAW A LINE AGAIN DIFFERENT
DEMENTIAS.
INCLUDING ALZHEIMER'S DISEASE
RELATED DEMENTIAS IN THE PLAN
AND ADRD ARE LEWY BODY AND
VASCULAR CONTRIBUTIONS TO
COGNITIVE IMPAIRMENT AND
COMBINATIONS AND WITH THE
NATIONAL INSTITUTE ON AGING
WE'RE TRYING TO REACH THE FIRST
GOAL OF THE NATIONAL PLAN AND
THAT IS TO PREVENT AND TREAT
ADRD.
CONGRESS AND THE AMERICAN PEOPLE
HAVE BEEN SUPPORTIVE OF THIS
EFFORT I'M SHOWING 2015 VERSION
US 2023.
2015 IS A GOOD BENCHMARK BECAUSE
OF UPDATES TO OUR SCIENTIFIC
CODING AND YOU CAN SEE THERE'S
BEEN A SIXFOLD INCREASE IN
SUPPORT FOR ADRD FUNDING AND
ADRD, SEVENFOLD INCREASE IN THAT
TIME.
THIS LOOKS SPECIFICALLY AT THE
ADRD AWARD FUNDING BY PHYSICAL
YEAR.
THE BACK PART INDICATES MONEY
THAT SUPPORTS ADRD RESEARCH.
THIS HAS BEEN PART OF OUR
MISSION AND CONTINUES TO BE SO
IT'S NOT SURPRISING THAT WE
WOULD FUND ADRD FROM OUR BASE
BUDGET AND CONGRESSIONALLY
DIRECTED FUNDS WE SPEND 10% OF
WE HAVE THIS ADDITIONAL MONEY IN
GRAY BARS THAT SUPPORT ADRD
RESEARCH AND ABLE TO FUND
MERITORIOUS PROJECTS THAT WOULD
NOT OTHERWISE BE ABLE TO BE
SUPPORTED BY BASE FUNDS AND
THERE'S TWO AWARDS.
ONE ON THE RIGHT YOU CAN SEE WE
HAVE INVESTIGATOR INITIATED
RESEARCH AND THE ADRD SPACE AND
LAST YEAR WE SUPPORTED ABOUT
$100 MILLION FOR THAT AND THEN
NUMBER TWO, WE HAVE RESEARCH
RESPONSIVE TO ADRD INITIATIVES
CREATED BY OUR PROGRAM STAFF
LAST YEAR ABOUT $200 MILLION IN
SUPPORT.
THE TOTAL AMOUNT FOR OUR
PORTFOLIO LAST YEAR OVER $440
MILLION.
THAT'S INCLUSIVE OF FUNDS AND
CONGRESSIONALLY DIRECTED FUNDS.
YOU CAN SEE BY DISEASE CATEGORY
HOW THAT IS SPENT ON AD AND FTD
AND AD IS A SIGNIFICANT FUND WE
HAVE AD IN OUR PORTFOLIO AND
CONVERSELY ADRD IN THEIR
PORTFOLIO.
IT'S VERY MUCH A TEAM EFFORT.
INSTEAD OF LOOKING AT THE
DOLLARS YOU CAN LOOK AT THE
NUMBER OF AWARDS.
I POINT YOU TO THE COLUMN ON THE
FAR RIGHT AND LEFT OF THAT.
THOSE ARE THIS NUMBER OF AWARDS
SINCE 2016 WE'VE GIVEN IN
DIFFERENT CATEGORIES OF ADRD.
NOT GOING TO GO THROUGH IT LINE
BY LINE BUT TO THE RIGHT TO
HIGHLIGHT NUMBERS, SINCE 2016,
WE'VE HAD 108 ADRD INITIATIVES.
FUNDING OPPORTUNITIES FOR THE
RESEARCH THAT RESULTED IN 436
AWARDS.
AT THE SAME TIME WE'VE HAD MORE
THAN 1,000 INVESTIGATOR
INITIATED AWARDS FOR ALMOST 100
NEW ADRD NINDS AWARDS SINCE
2016.
NOW, AN IMPORTANT METRIC IS NOT
JUST THE DOLLARS AND GRANT
NUMBERS ABOUT THE WE WANT TO
KNOW THE SCIENCE IS MOVING
FORWARD.
THIS MONEY ALLOWS US TO ADDRESS
GAPS IN THE FIELD AND WE'RE ABLE
TO DEDICATE RESOURCES TO ADDRESS
THOSE GAPS.
I WANT TO TALK ABOUT THE PROCESS
AND SOME OF OUR SUCCESS.
EVERY THREE YEARS WE HAVE THESE
SUMMITS FOR OUR ADRD RESEARCH
PRIORITIES AND FROM THESE
SUMMITS COME RECOMMENDATIONS AND
MILESTONES.
IT'S BEYOND THE SCOPE TO DISCUSS
ALL THOSE TODAY BUT WE CAN LOOK
AT THE OVERARCHING THEMES FROM
THE RECOMMENDATIONS AND
MILESTONES.
HERE'S SOME BULLETS.
I WON'T READ THEM ALL BUT HAVE
WORDS HIGHLIGHTED.
HEALTH EQUITY, PRAGMATIC
APPROACHES ESPECIALLY PRAGMATIC
CLINICAL TRIALS, PRECISION
BIOMARKERS, PERSONALIZED
MEDICINE, PREVENTION, VIGILANCE,
NOVEL STRATEGIES AND TOOLS ARE
CROSS-CUTTING THEMES ACROSS OUR
SUMMIT IN 2022.
KNOWING THESE THEMES THAT ARE
IMPORTANT WHEN WE COUNTER
SPECIFIC GAPS, THIS PROBLEM
DEMENTIA IS OFTEN UNDIAGNOSED IN
PRIMARY CARE AND WHEN IT IS
UNDIAGNOSED IT GOES UNTREAT AND
IS A PROBLEM IN OLDER, BLACK AND
HISPANIC PATIENTS BEING UNDER
DIAGNOSED.
WE ARE ABLE TO CREATE FUNDING
OPPORTUNITIES BASED ON THIS
INFORMATION.
SO THE FUNDING OPPORTUNITY
NUMBERS ARE ON THE BOTTOM LEFT
OF THE SLIDE.
WE'RE CALLING FOR PRAGMATIC
CLINICAL TRIALS TO DETECT
COGNITIVE IMPAIRMENT AND
DEMENTIA.
PROGRAM STAFF CREATED THIS.
AS A RESULT WE GOT MERITORIOUS
APPLICATIONS FOR DETECTING
COGNITIVE DEMENTIA AND THE
PARADIGMS DIFFER BUT THEY'RE ALL
RAPID ASSESSMENTS.
THEY DON'T REQUIRE A LOT OF
STAFF TRAINING AND CAN BE DONE
IN THE REAL WORLD.
I'LL HIGHLIGHT ONE CALLED THE
5-COG PROVEN TO BE SUCCESSFUL.
5 REPRESENTS THE ASSESSMENT CAN
BE DONE IN FIVE MINUTES IT'S A
FAIR DETECTION TOOL THAT USES
SIMPLE MATCHING AND THIS WAS A
RANDOMIZED TRIAL AND URBAN CARE
WITH THOSE OF COGNITIVE CONCERNS
AND THE DEMENTIA AND CARE
ASPECTS OF THE STUDY SHOWING A
SIGNIFICANT IMPROVEMENT IN
DETENTION AND EARLY TREATMENT.
WHAT WE HAVE IS AN
EVIDENCE-BASED APPROACH THAT'S
EQUITABLE FOR DETECTING
COGNITIVE IMPAIRMENT AND
DEMENTIA AND CAN BE IMPLEMENTED
IN THE REAL WORLD.
>> PEOPLE WITH PROBABLE
ALZHEIMER'S DEMENTIA AND THE
PATHOLOGY IS TYPICALLY MIXED.
IT'S NOT AN ARCHTYPEAL PATHOLOGY
AND MIXED PATHOLOGY IS THE RULE
NOT THE EXCEPTION.
SO IN TALKING ABOUT MIXED
PATHOLOGY SINCE WE WANTED TO
TREAT INDIVIDUALS AND THEY MAY
PRESENT SIMILARLY BUT THE
PRESENTATION MAY HAVE COMPLEX
MIXED PATHOLOGY WE NEED
BIOMARKERS TO UNDERSTAND THE
DISEASE PROCESS IN THE PEOPLE
AND TO BETTER STRATIFY AND
INFORM OUR CLINICAL TRIALS 37
WE'VE BEEN SUPPORTING BIOMARKER
DEVELOPMENT AND WE'VE FOUND GOOD
BIOMARKERS FOR ALZHEIMER'S
PATHOLOGY AND ESPECIALLY THE P
TAU BIOMARKERS.
THIS IS AN AREA WE'VE BEEN
FUNDING AND SEEK CONTINUED
DEVELOPMENT IN.
P TAU 217 IS A GOOD PROGNOSTIC
BIOMARKER AND COGNITIVELY
UNIMPAIRED ADULTS AND HAS A SEX
SPECIFICITY WITH AN INDICATOR OF
DECLINE IN WOMEN THAN IN MEN.
THE PAPERS HIGHLIGHT THE
IMPORTANCE OF LOOKING AT P TAU
217 RATIO TO UNPHOSPHORYLATED
TAU.
THEY CAN STREAMLINE GETTING
TREATMENT GROUPS BECAUSE OF HOW
THEY MIRROR AMYLOID PET POSITIVE
RESULTS.
KIDNEY DYSFUNCTION CAN HAVE AN
EFFECT IN TAU LEVELS IN BLOOD.
IF THE RATIO TO P TAU TO
UNPHOSPHORYL ATED TAU IS USED IT
CAN BE USED IN A BIOMARKER IN A
COHORT WITH VARIOUS LEVELS OF
KIDNEY DYSFUNCTION USING THE
RATIO INSTEAD OF P-TAU LEVELS.
AND P TAU 181 CAN BE USED TO
IDENTIFY PATIENTS WITH
CO-PATHOLOGY.
AGAIN, HELPING STRATIFY CLIN
KRAL TRIALS.
AND WE'VE BEEN ENTHUSE --
ENTHUSIASTIC AND THERE'S A
PRIMARY FUNCTIONAL FORMS IN THE
CYTOPLASM.
YOU GET A LOSS OF FUNCTION.
IT'S NORMALLY INVOLVED IN
SPLICING.
YOU GET THE INCLUSION OF CRYPTIC
EXONS.
TRANSCRIPTS ARE PRODUCED WITH
THE EXONS.
FOR THE MOST PART AND THE RNAs
TEND TO BE LESS STABLE BECAUSE
OF DECAY BUT THE TRANSCRIPTS CAN
BE BIOMARKERS BUT IN SOME CASE
THE CRYPTIC EXON IS IN FRAME SO
THEY CODE AND YOU GET A PROTEIN
THAT HAS A CRYPTIC PEPTIDE
WITHIN THE PROTEIN.
AND DISCOVER ANTIBIOTICS FOR
DETECTION AND WITH THE PROTEIN
THE CRYPTIC EXON IS IN FRAME SO
A PROTEIN IS EXPRESSED AND
ANTIBODIES THE CRYPTIC PEPTIDE
CAN BE USED IN NINDS SUPPORTED
RESEARCH TO SEE THIS IN PLAIN
TISSUE AND PLASMA AND THIS IS
EXCITING.
THIS DEVELOPMENT BECAUSE IT'S
HIGHLY SPECIFIC FOR TBD43
PATHOLOGY.
WE DON'T EXPECT TO SEE THESE
CRYPTIC PEPTIDES IN HEALTHY
INDIVIDUALS.
THIS IS SOMETHING WE'RE
SUPPORTIVE OF AND WILL CONTINUE.
WE SUPPORTED MANY PROJECTS AND
TECHNOLOGIES IN THIS AREA OF
AMPLIFICATION ASSAYS AND THE
PROTEIN CAN HAVE A PCR REACTION
AND THE LARGER AGGREGATES TO BE
DETECTED BY FLUORESCENT DYES FOR
DETECTING PATHOLOGY FROM CSF.
HERE'S A COHORT WITH ALZHEIMER'S
WITH A THIRD BEING DETECTED IN
SUCH A COHORT THAT'S INFORMATIVE
FOR STRATIFYING OUR CLINICAL
TRIALS AND DIRECTING TREATMENT
TO THE UNDERLYING DISEASE.
WHAT WE'RE ABLE TO DO IS SET
OUR CLINICAL TRIALS FOR
TEMPERATURE AND EFFECTIVE
TREATMENTS.
I'LL TURN IT OVER TO AMBER.
>> THANK YOU, FRANK.
THAT SEEMS LOUD.
I'M GOING TO WRAP UP THE ADRD
UPDATE TALK BEING ABOUT THE NEXT
ADRD SUMMIT THAT IS START BEING
TO BE AND IS KICKING OFF RIGHT
NOW.
I'M AN ADRD SCIENTIFIC PROJECT
MANAGER AT NINDS.
THE NEXT SUMMIT WILL BE MARCH 25
AND 26 THIS IS A SAVE THE DATE
SO PLEASE NOTE THESE DATES AND
THE MORE INFORMATION WILL BE
COMING ZOON AND WE'RE THRILLED
TO WORK WITH UCSF AND THE
SCIENTIFIC CHAIR AND DR. POSSIN
IS PART OF EFFORTS TO ADDRESS
DEMENTIA IN EVERYDAY CLINICAL
SETTING.
BECAUSE THE SUMMITS ARE SO
IMPORTANT FOR THE PROGRAM I WANT
TO SPEND MORE TIME EXPLAINING
ABOUT THE SUMMITS AND WHAT WE
ARE DOING.
WE HEARD ABOUT THE NINDS
COLLABORATION OF THE ASPECTS
INCLUDING THE SUMMITS TOGETHER
SHAPE NATIONAL RESEARCH
PRIORITIES FOR ALZHEIMER'S
DISEASE AND ALZHEIMER'S DISEASE
RELATED RESEARCH.
THERE ARE THREE SUMMITS.
NIA LEADS THE SUMMIT AND THE
NEXT IS IN A FEW WEEKS.
YOU COULD FIND IT ONLINE.
I DON'T HAVE THE DETAILS HERE.
THEY ALSO LEAD THE CARE SERVICES
AND SUPPORT SUMMIT WHICH
HAPPENED LAST YEAR RECENTLY AND
NINDS LEADS THE ALZHEIMER'S
DISEASE RELATED DEMENTIA SUMMIT.
THE TOPICS OF THE SUMMITS HAVE
BEEN OVER ARCHING INCLUDING
MIXED DEMENTIAS AND
NOMENCLATURE.
IN 2022 WE HAD A SESSION ON THE
IMPACT MUCH COVID-19 IN AD AND
RD.
THERE'S DISEASE SPECIFIC
SESSIONS ON CONTRIBUTION TO
COGNITIVE IMPAIRMENT AND
DEMENTIA AND AD RD SESSIONS
CLUCKED ON
SESSIONS ON TD43 AND THE SUMMIT
TAKES PLACE OVER ABOUT A
SEVEN-MONTH PERIOD WITH THINK
TANK STYLE PROCESS WHERE
COMMITTEES MEET AND RE-EVALUATE
THE MILESTONES THAT ARE EXISTING
AND COME UP WITH DRAFT RESEARCH
RECOMMENDATIONS PRESENTED AT THE
ADRD SUMMIT FOR INPUT.
THE RESEARCH RECOMMENDATIONS
BECOME PART OF THE NATIONAL
PLAN.
AND IN 2022 THERE WERE 52 ADRD
RESEARCH MILESTONES IN THE
NATIONAL PLAN.
THIS IS AN ORGANIZATIONAL
STRUCTURE HOW WE APPROACH THE
SUMMIT.
I'M SHOWING HERE FOR THE 2022
SUMMIT WHY WILL TAKE THE SAME
STRATEGY NOW WITH 2025 SUMMIT
THE MILESTONES THAT WERE
EXISTING FROM THE 2019 SUMMIT
WERE TAKEN UP BY SEVEN DIFFERENT
COMMITTEES THAT RE-EVALUATED THE
MILESTONES TO MAKE UPDATES ON
THE ANALYSES DISCUSSION AND FOR
CONSIDERATION OF SCIENTIFIC
ADVANCES AND WE HAD A NEW
SESSION ON COVID-19 THAT WAS AN
EMERGING TOPIC THAT WAS COVERED
DURING THE SUMMIT.
THESE WERE PRESENTED AS DRAFT
RECOMMENDATIONS AND BEFORE THEY
WERE FINALIZED.
THE FINALIZED MILESTONES YOU CAN
READ IN THE REPORT WITH THE QR
CODE.
ALL THE MILESTONES IN THE
RECOMMENDATIONS IN THE REPORT
REPRESENT IMPORTANT ADRD
PRIORITIES.
BECAUSE OF THE APPROACH WE TAKE
A FACT OF CONSENSUS BUILDING
PROCESS THESE ARE NATIONAL
PRIORITIES THAT REPRESENT THE
TOP GOALS IN THE FIELD.
AND THE ADRD BUDGETS AND
CORRESPONDING BUDGETS AS THEY
BECOME AVAILABLE FROM CONGRESS.
I'LL END WITH SOME OF THE
DETAILS WITH ALL OF THE OPEN
NINDS-LED ADRD FUNDING
OPPORTUNITIES AND A COUPLE
APPROVED CONCEPTS.
YOU DON'T HAVE TO REMEMBER ALL
OF THIS.
THERE'S ALL THE FUNDING
OPPORTUNITIES ARE ON OUR WEBSITE
AND I'LL GIVE A QR CODE AND LINK
IN A COUPLE SLIDES.
THERE ARE THREE TRAINING FUNDING
OPPORTUNITIES.
TWO K99s AND ADMINISTRATIVE
SUPPLEMENT PROGRAM.
WE HAVE EIGHT DISEASE MECHANISMS
FUNDING OPPORTUNITIES AND SOME
ARE FOR VASCULAR DEMENTIA FOR
LEWY BODY DEMENTIA AND SOME ARE
CROSS-CUTTING.
THERE'S ONE RESEARCH RESOURCE
AND FOUR CLINICAL TRIAL FUNDING
OPPORTUNITIES.
THE OF THE OF THE INVESTIGATOR
INITIATED RESEARCH IS WELCOME
AND THERE'S NO SPECIAL FUNDING
OPPORTUNITY NEEDED TO APPLY FOR
THE CONSIDERATION FOR THE
SPECIAL PAY LINE FOR ALL
SERVICES RELATED TO DEMENTIA
GRANTS.
SO YOU CAN COME IN UNDER THE
PARENT RO1 AND SPECIAL PAY LINE
APPLIES.
WE TALKED HOW THE NINDS ADRD
PROGRAM HOPEFULLY GUIDES
RESEARCH AND CONTRIBUTES TO NEW
ADVANCES AND ADVANCED
UNDERSTANDING TOWARDS
INTERVENTIONS.
WE ISSUED MORE THAN 100 FUNDING
ANNOUNCEMENTS AND THERE'S ABOUT
15 MORE IN PHYSICAL '25 INVITING
APPLICATION TO FILL CRITICAL
NEEDS.
WE'VE SEEN FOR THE ALZHEIMER'S
DISEASE RELATED FUNDING WE'VE
SEEN AN INCREASE OF MORE THAN
SEVEN FOLD SINCE 2015.
MARCH 25, 26 IS THE SUMMIT AT
NIH AND ALSO ONLINE.
HERE'S THE QR CODE FOR OUR WEB
PAGE WHERE WE KEEP A LIST OF THE
APPROVED FUNDING CONCEPTS AND
PUBLISHED FUNDING ANNOUNCEMENTS
AND YOU CAN GO TO THE SITE OR
E-MAIL TO JOIN THE LIST SERVE
AND GET E-MAILS AS THE
ANNOUNCEMENTS BECOME PUBLISHED
WE'LL SHARE THEM WITH YOU.
AND I'M GOING TO GO TO A FINAL
SLIDE THAT SHOWS THE SPECIAL
SIZE OF THIS PROGRAM.
WE WORK WITH ALMOST EVERY
PROGRAM DIRECTOR AT NINDS AND WE
WERE SO GRATEFUL FOR THE TEAM
EFFORT THAT WE HAVE HERE.
NINDS AND LEADERSHIP AND STAFF
WORK TOGETHER.
PEOPLE IN THE BOLD ARE THE
COORDINATING UNIT MEMBERS AND
WE'RE GRATEFUL FOR THEIR EFFORTS
AND EVERYONE ELSE LISTED HERE
AND I'M SURE I MISSED SOME
PEOPLE WHO WORKED ON ALL ASPECTS
OF THESE TOGETHER.
IF I CAN GO BACKWARDS -- TOO
FAR.
PUT BACK THE QR CODE TO GO TO
OUR WEBSITE WITH THE CONTACT
INFORMATION AND FUNDING
ANNOUNCEMENTS.
WE'RE HAPPY TO TAKE QUESTIONS.
I CAN ALSO SHARE PDF OF THE
SLIDES SO PLEASE DO ASK.
>> I THINK WE GIVE THEM THE
SLIDES.
JUST BEFORE WE GET TO THE
QUESTIONS I WANT TO PUT THIS IN
CONTEXT.
WE HAVE THESE THREE
CONGRESSIONALLY MANDATED
PROGRAMS, ADRD AND HEAL AND
BRAIN AND SINCE THEY INVOLVE
CONSIDERABLE MONIES WE LIKE TO
COME IN AND DO THIS SO FOLKS
KNOW.
>> HOW ONE CONTINUES TO JUSTIFY
IN THE PARKINSON'S RELATED
DEMENTIA WHEN YOU SHOWED OVERLAP
IN COMORBIDITY AND DIFFERENT
OPATHIES.
>> THOSE RELEVANT TO PARKINSON'S
DISEASE ARE FREQUENTLY COVERED.
THERE'S BASIC BIOLOGY AND
MECHANISM WE DO FUND.
>> LEWY BODY DEMENTIA INCLUDES
PARKINSON'S DEMENTIA AND LEWY
BODY.
>> I THINK WHEN YOU HAVE
SUCCESSES YOU NEED TO TALK ABOUT
IT.
I THINK THE SUCCESS IN
BIOMARKERS IS EXTRAORDINARY.
THE WAY PLASMA-BASED BIOMARKERS
HAVE ALREADY CHANGED THE GAME
CLINICALLY IS EXTRAORDINARY.
AS MARKERS AND SITUATION MARKERS
MAKE THEIR WAY IN THE SPACE THE
ABILITY TO HAVE PRECISION
DIAGNOSIS MAKING AN IMPACT IS
HUGE.
A GREAT SUCCESS.
>> THANK YOU FOR THE FEEDBACK.
AND WE'LL WORK ON SELLING IT
BETTER BECAUSE I'M EXCITED TOO.
>> I TAUGHT WHITE MATTER
INTENSITIES WERE FRECKLES AND
I'LL EXCITED WHERE WE'RE HEADED
SO CONGRATULATIONS.
>> I WOULD UNDER SCORE THE
PROGRESS IN THE BIOMARKER
DEVELOPMENT.
THE DATA JUST SHOWN RECORDING
SPLICING AND ASSESSMENT OF THE
ASSESSMENT ARE TRANSFORMATIONAL
IN ALS AND ALS RELATED FTD.
IT ILLUSTRATES ANOTHER POINT
WHICH IS HOW IMPORTANT IT IS TO
GET BRILLIANT INVESTIGATORS IN
THE FIELD AND REFLECTS THE
RECRUITMENT OF MICHAEL WARD WHO
JUST HAD A HUGE IMPACT AND MARKS
THE START OF A NEW TYPE OF
BIOMARKER AND WE'RE ALL GRATEFUL
FOR THAT.
>> WE'RE A TOUCH OVER BUT WE
CHECKED WITH OUR NEXT SPEAKER
AND WE'LL BE STARTING AT 1:15
INSTEAD OF 1:00 AND EVERYTHING'S
OKAY, RIGHT, TIM?
OKAY.
WITH THAT I WANT TO THANK
EVERYONE FOR THE MORNING SESSION
AND WE'RE ADJOURNED FOR LUNCH
AND SEE YOU BACK AT 1:15.
PLEASURE TO INTRODUCE
FYODOR URNOV AT THE INNOVATIVE
GENOMICS INSTITUTE AND PIONEER
IN THE THERAPEUTIC FIELD OF
GENOME EDITING, ADVANCING
TECHNOLOGIES FOR GENOME EDITING
AND PUSHING TECHNOLOGIES TO HOW
IT CAN SOLVE REAL-WORLD PROBLEMS
AND HE WAS ABLE TO REALLY MAKE A
GREAT CONTRIBUTION IN OUR
GENETICS AND GENOMICS AND
EPILEPSY MEETING AND ASKED HIM
TO COME AND TALK ABOUT THE
GENOMICS INSTITUTE AND THE
RECENT REPORT THEY PUT OUT IN
HOW WE CAN SCALE UP TECHNOLOGIES
FOR NEUROLOGICAL DISORDERS AND
THERE ARE HUNDREDS OF THEM AND
LOOKING TO THE FUTURE.
AS I SAID BEFORE THE USUAL SMALL
MOLECULE COMMERCIAL MODEL MAY
NOT BE WHAT WE NEED AND THERE
MIGHT BE OPTIONS THAT ARE BETTER
BUT HOW TO GET THOSE INTO
PRACTICE IS STILL EARLY STAGE.
I KNOW FYODOR AND THE IGI GROUP
HAVE BEEN THINKING ABOUT THIS
AND WANTED TO HEAR THEIR
THOUGHTS.
IT'S A REAL PLEASURE TO HAVE YOU
AND LET'S GET INTO IT.
>> THANK YOU, DR. KOROSHETZ.
IT'S A HUMBLING EXPERIENCE TO
REPRESENT TWO INSTITUTIONS AT
THE INNOVATIVE GENOMICS
INSTITUTE I'M IN AND OF COURSE
THE UNIVERSITY OF CALIFORNIA.
THIS WILL NOT BE A MERE UPDATE
IN THE CRISPR SPACE, AND I'LL
ARGUE WE'RE IN A UNIQUE POSITION
TO HAVE THE FEDERAL SECTOR MAKE
AN ENABLING IMPACT ON THE ACCESS
TO GENETIC MEDICINES FOR GENETIC
DISEASES AND NEWER GENETIC
DISEASES ARE AT THE FRONT OF AND
I THINK NINDS IS IN A UNIQUE
POSITION TO CONTINUE TO MAKE THE
IMPACT IT HAS AND HAVE THE
IMPACT GROW.
I'LL CLOSE IN ABOUT 15 MINUTES
WITH ACTIONABLE NEXT STEPS.
THEY'LL BE NEXT STEPS.
THE WORK BEGAN IN 1994 IN 1995
WITH MY COLLEAGUES SHOWED IT CAN
WORK FOR THERAPEUTIC PURPOSES
AND THEY INVENTED CRISPR GENE
EDITING AND IN 2023 WE HAVE OUR
FIRST CRISPR APPROVED MEDICINE
FOR SICKLE CELL DISEASE AND
VICTORIA GRAY WAS THE FIRST
HUMAN BEING ON THE TRIAL.
UNFORTUNATELY, AND I KNOW I
DON'T WANT TO BE A DEBBIE DOWNER
BUT I WANT TO BE DEBBIE REALIST,
DESPITE THAT MARVELOUS ADVANCE
THE CRISPR POTENTIAL TO TREAT
GENETIC DISEASE IS UNDER THREAT
AND TELL YOU WHAT THAT IS AND
WHAT THE FEDERAL GOVERNMENT AND
SPECIFICALLY THE NIH AND NINDS
CAN DO TO MAKE A DIFFERENCE.
WE GET E-MAILS LIKE THIS I GET
ONE OR TWO A DAY.
THEY SHARED WORK WITH MY SISTER,
MARY.
SHE HAS A RARE GENETIC MUTATION
AND HER SISTER IS INTERESTED
WHETHER SHE COULD BE A SUBJECT
FOR CRISPR.
UNDER THE STATUS QUO THE CHANCES
OF THIS HUMAN BEING RECEIVING A
THERAPY BEFORE SHE LOSES HER
MOTOR FUNCTION THE CHANCES OF
GETTING AN ATTEMPT WITH CRISPR
IS ZERO AND NOTHING TO DO WITH
THE TECHNICAL ISSUES, THEY'RE
LOGISTICAL AND COMMERCIAL.
THIS IS WHERE THE FEDERAL SECTOR
CAN PLAY A UNIQUE ROLE AS I'M
ABOUT TO EXPLAIN.
OVER THE NEXT FEW MINUTES I'LL
TELL YOU DRIVERS OF PROGRESS OF
CRISPR FOR RARE DISEASES COME
FROM SIX INTERLOCKED COMPONENTS.
THE PUBLIC SECTOR EFFORT
CONNECTED TO PUBLIC-PRIVATE
PARTNERSHIPS WHERE THE TOOLBOX
OF EDITING ARE IMPROVED AND
CREATES A TRACK RECORD WITH APPS
TO LICENSURE AND APPROVAL.
I'LL WALK THROUGH EXAMPLES.
LET'S START WITH THESE THREE AND
START WITH A FLAGSHIP PROGRAM ON
PLANET EARTH FOR CRISPR CURES.
THERE'S NO OTHER PROGRAM LIKE
THIS ANYWHERE IN THE WORLD AND
HAPPENS TO BE OUT OF THE NINDS
AND THE SOMATIC CONSORTIUM AND
FIVE TEAMS IN THE UNITED STATES
HAVE BEEN FUNDED BY THE NIH WITH
NINDS AND DR. KOROSHETZ BEING
THE LEAD AND M CATS FOR YOUR
STEWARDSHIP ON THE PROGRAM.
FIVE ACADEMIC TEAMS HAVE BEEN
FUNDED TO TAKE CRISPR CURES TO
THE CLINIC IN PLATFORM MODES SO
IT'S A RISING TIDE THAT LIFTS
OTHER BOATS.
I'LL REVIEW WHAT THEY'RE ABOUT
TO GIVE YOU A FLAVOR OF WHAT WE
CAN EXPECT FROM THIS
FEDERALLY-FUNDED EFFORT AND HOW
TO LEVERAGE AND EXPAND THE
FOOTPRINT.
THE GOME IS TO CREATE A DOUBLE
STRAND BREAK TO TREAT HUNTING
TONS DISEASE AND ALS.
HOW DO YOU USE A DOUBLE-STRAND
BREAK FOR THAT?
I'LL WALK YOU THROUGH WHAT'S
BEEN USED CLINICALLY AND GIVE A
SENSE WHAT WE'RE TRYING TO DO
AND WHAT THE EXPANSION OF THE
EFFORT COULD LOOK LIKE.
AS INVENTED, THE REMARKABLE KAS
9 CAN BE ROUTED BY BEING ARMED
WITHIN AN RNA MOLECULE,
NUCLEOTIDE THE LOCATION CODE
WHERE BASE PAIRING WILL BE ABLE
TO FIND ITS TARGET AND CREATE A
DOUBLE-STRAND BREAK.
YOU GET INSERTIONS AND DELETIONS
AND THAT'S THE MODALITY FURTHEST
ALONG AND GENE EDITING IS A
DIFFERENT PROCESS AND IT'S A
DOUBLE-STRAND BREAK REPAIRED BY
INSERTIONS AND DELETIONS.
THERE'S DOUBLE-STRAND BREAK
EDITING IN VIVO.
INTELIA TREAT AMYLOIDOSIS TO
DELIVER AN mRNA KAS 9 AND
DISRUPT TTR.
MECHANISTICALLY SPEAKING IS THE
CELL LEVEL ONCE YOU ADMINISTERED
THE TWO COMPONENTS IS ONCE THE
LIPID NANO PARTICLE IS UP TAKEN
BY THE LIVER IT FORMS KAS 9,
THAT BINDS AND THAT GUIDES THE
ENZYME TO THE TTR GENE AND WE
HAVE A KNOCKOUT OF THE TOXIC
PROTEIN.
HOW WELL IS THIS WORKING?
EVEN AS I RIGHT THE MOST ROSY
UTOPIA IN THE UNIVERSE I COULD
NOT HAVE PREDICTED HOW WELL THIS
HAS GONE.
THIS GIVES A SUMMARY SHORTLY AND
YOU'RE LOOKING AND THE Y AXIS IS
REDUCTION POST DOSING OF THE
SERUM BIOMEDICAL OF DISEASE
EFFICACY.
SO LESS IS BETTER AND THE LOW
DOSE COHORT OF CHRIS PER KAS.
THERE'S A LOVELY DOSE EFFECT
WHERE AT THE HIGHEST DOSE COHORT
WITH NO NOTICEABLE TOXICITIES WE
GET 93% KNOCKOUT OF THE GENE.
THE HUMAN LIVER IS AN OCCUR AN
-- ORGAN AND WE KNOCKED OUT THE
COPY OF THE GENE.
INTELIA HAS DONE SO WELL AND SO
WELL TOLERATED THEY STARTED A
PHASE III TRIAL WITH 765
SUBJECTS.
THIS IS PROBABLY GOING TO BE THE
NEXT APPROVED CRISPR MEDICINE.
ONE OF THE THREE MOST IMPORTANT
SLIDES I'M ABOUT TO SHOW YOU IS
COMING UP.
AS YOU KNOW IN GENETIC THERAPIES
YOU WANT TO ESTABLISH SAFETY
FIRST AND SUBJECTS ON THE LOWER
DOSE COHORT SOME REDUCTION BUT
THIS IS NOT ENOUGH FOR
THERAPEUTIC BENEFIT.
IF THIS WERE A STANDARD GENE
THERAPY WITHOUT AN ASSOCIATED
VIRUS THAT WOULD BE IT.
YOU CAN'T DOSE THE SUBJECT AGAIN
BECAUSE OF IMMUNE ISSUES.
THE KAS 9 mRNA IS SO SHORT
THEY'VE BEEN ABLE TO
SUCCESSFULLY ABLE TO REDOSE THE
LOW-DOSE SUBJECTS AND GET THEM
INTO THE THERAPEUTIC EFFICACY OF
90%.
IT CAN BE TAPERED IN.
SO THIS MEANS IT'S A UNIQUE
GENETIC MEDICINE.
YOU CAN DOSE TO EFFECTS.
MY BIGGEST FEARS ARE OFF
TARGETS, TOXICITIES, CRISPR CAN
BE CLINICALLY ADMINISTERED AT
THE LOW DOSE AND YOU WAIT AND
WAIT UNTIL YOU DOSE TO EFFECT.
THIS GIVES A UNIQUE OPPORTUNITY
TO HAVE CRISPR MAKE AN IMPACT ON
YOUR GENETIC DISEASE BECAUSE YOU
CAN START SLOW AND ACCELERATE
BASED ON HOW SUBJECTS ARE DOING.
THIS IS A COMPONENT OF WHAT
WE'RE FUNDED TO DO.
MY COLLEAGUE INVENTED AN RNP
PROTEIN PARTICLE TO DELIVER THIS
TO A BREAK.
AND THE GOAL HERE IS TO CREATE
TWO DOUBLE STRAND BREAKS THAT
WILL EITHER EXCISE THE REPEAT IN
HUNTINGTON OR IN ALS.
I'M EXCITED THIS IS IN PART A
VIRAL APPROACH.
AS I SHOWED EARLIER THERE'S
CLINICAL PROOF OF CONCEPT FOR
REDOSING.
AS WE ADVANCE THIS TO IND AND
HIS HOPES TO GO TO THE CLINIC IN
FOUR YEARS WE'RE EXCITED TO HAVE
THE CLINICAL DEVELOPMENT PLAN IN
SUCH A LOW DOSE AND THEN TAPER
UP BASED ON HOW THE SUBJECT IS
DOING APPROACH.
TO BE CLEAR, THIS IS A SPECIFIC
EXAMPLE OF TWO SPECIFIC
DISEASES.
THERE ARE PRINCIPLES OF START
LOW AND GO HIGH WITH NON-VIRAL
CRISPR AND THE TOOLBOX OF CRISPR
WAS DEVELOPED 37 THE NEXT
PROJECT USES AN ELABORATION OF
GENE EDITING INVENTED CALLED
BASE EDITING AND DOESN'T INVOLVE
A DOUBLE-STRAND BREAK.
YOU CAN CORRECT POINT MUTATIONS.
WHY IS THAT USEFUL?
YOU CAN MAKE A POINT MUTATION
THAT INACTIVATES THE SPLICE
BEING OF A GENE AND 15 HUMAN
BEINGS HAVE BEEN DOSED FOR THEIR
FAMILIAL CHOLESTEROLEMIA AND
THERE'S SUBJECTS ON LOW, MEDIUM
AND HIGH DOSE COHORTS AND
THEY'RE KNOCKING OUT THIS AND
THOUGH IT WAS DOSED AT THE
EARLIER TIME-OUT THE EFFECT TO
60% REDUCTION AT THE HIGH DOSE
COHORT WE'RE USING IT IN VIVO
AND THE SAME DELIVERY PLATFORM.
I'LL USE THIS WORD A LOT TODAY.
PLATFORM MEANS PLUG AND PLAY.
REPLACE TOPPINGS ON A COOKIE OR
PIZZA.
BY THE SAME TOKEN YOU CAN USE
THE SAME FRAMEWORK AND THE BASE
EDITOR AND GUIDE RNA BUT THE
ROUTE OF ADMINISTRATION IS THE
SAME AND NON-CLINICAL PATH IS
SIMILAR.
IT'S THIS PLATFORM NATURE OF
CRISPR TO BUILD A NEW MEDICINE
THAT I THINK IS AT THE
CENTERPIECE OF THE PLACE WHERE
THE FEDERAL GOVERNMENT COULD
MAKE AN IMPACT.
WITHIN THE SOMATIC CONSORTIUM A
TEAM LED BY JACKSON LAB AND
HARVARD ARE USING THIS TO TREAT
SMA.
SO ONE LAB DEVELOPED A BASE
EDITOR THAT WILL PROVIDE A GENE
EDITING APPROACH TO TREAT SPINAL
MUSCULAR ATROPHY IN TERMS OF HOW
THEY CAN USE AD TO DELIVER AND
CREATE THE DESIRED MUTATION AND
MOST IMPORTANTLY HOW THEY HAVE A
BENEFIT ON ANIMAL SURVIVAL.
IT'S EXCITING THEY'RE TAKING
THIS TO THE CLINIC UNDER FEDERAL
FUNDING BECAUSE ALL THE
LEARNINGS WILL BECOME PART OF
THE FEDERAL GOVERNMENT'S
KNOWLEDGE BASE.
FINALLY, IN THE INTEREST OF TIME
I'LL DO THIS QUICKLY.
THE BROAD HAS BEEN FUNDED TO
TAKE ANOTHER MODALITY OF
RNA-BASED APPROACHES TO THE
CLINIC CALLED EPIGENOME EDITING.
NO DOUBLE STRAND BREAKS OR POINT
MUTATION BUT LONG EFFECTS FROM
EXPOSURE DESIGN TRANSCRIPTION
FACTOR BASED ON TECHNOLOGY
DEVELOPED TWO DECADES AGO A
PROTEIN TARGETS A CHROMATIN DNA
REMODELER TO SILENCE THE PRION
PROTEIN AND THIS IS A FEDERALLY
FUNDED EFFORT.
SMA, CMS, WET SYNDROME AND PRION
DISEASE TO THE CLINIC.
I GIVE YOU THE PUBLIC SECTOR
EFFORT AND THE TOOLBOX OF
EDITING GROWTH AND TRACK RECORD.
I'D LIKE TO SPEAK TO DELIVERY.
THERE'S A TROPE IN MY FIELD, WE
WOULD DO MORE CRISPR IF ONLY
DELIVERY WORKED.
THAT EXCUSE IS RAPIDLY RUNNING
OUT OF STEAM.
DELIVERY FOR NEUROLOGIC DISEASES
IS MAKING REMARKABLE PROGRESS.
I WANT TO HIGHLIGHT TWO
DEVELOPMENTS.
THIS IS BEAUTIFUL WORK IN THE
RECENT PAPER WITH AN ADENOVIRUS
WITH SYSTEMIC ADMINISTRATION OF
AN AAV AND YOU SEE THIS
REMARKABLE COVERAGE OF THE MOUSE
AND SUBSEQUENTLY AN HP DATA ON
THEIR WAY AND THIS IS THE AAD
MODALITY THE EFFORT FOR PRION
DISEASE TAKING TO THE CLINIC
WHICH IS REMARKABLE.
I'M GOING TO TAKE 60 SECONDS TO
SHOW DATA FROM THE GENOMIC
INSTITUTE TO SHOW YOU WHAT
INNOVATION IS OUT THERE.
THIS IS WORK FROM AN
INVESTIGATOR IN THE MOLECULAR
CELL BIOLOGY DEPARTMENT USING
LIPID NANO PARTICLES AND
DEVELOPING LNP DELIVERY TO THE
BRAIN.
THIS IS DELIVERY OF THE BRAIN
ORGANOIDS AND WE'RE COMPARING
LENTI VIRUS DELIVERY OF CRE AND
I'LL DIM THE LIGHTS AND THEY'RE
MORE ROBUSTLY POSTED.
THEY'RE 9-MONTH-OLD HUMAN
ORGANOIDS.
WE HAVE EARLY DATA ROBUST ENOUGH
TO SHARE WITH THIS AUDIENCE TO
DO THIS FOR AN IN VIVO
ADMINISTRATION TO THE STRIATUM
AND I EMPHASIZE THIS IS NOT A
VIRUS, THIS IS LNP DELIVERY AND
USING EARLY COMPOUNDS UP TO 30%
OF THE STRIATUM WAS OBTAINED.
I WANT TO EMPHASIZE THIS IS NOT
DATA FROM THE IGI BUT NON-VIRAL
DELIVERY OF CRISPR TO THE
PERIPHERAL AND NERVOUS SYSTEM
WILL BE A CLINICAL REALITY.
WHEN THAT HAPPENS, EVERYTHING I
SAID EARLIER ABOUT DOSING TO
EFFECT WE HAVE TO INTEGRATE
CRISPR DEVELOPMENT TO WHEN
DELIVERY DOESN'T WORK.
THAT EXCUSE WILL BE JUST GONE
AND THAT FUTURE IS SOONER THAN
WE THINK.
I WANT TO SPEND THE LAST FEW
MINUTES OF THIS PREAMBLE BEFORE
GETTING TO THE ACTION ITEMS WITH
PUBLIC-PRIVATE PARTNERSHIPS AND
NOVEL PATHS TO LICENSURE.
PUBLIC-PRIVATE PARTNERSHIPS
FIRST.
I MENTIONED THE PERSON WITH A
SEVERE NEUROLOGIC DISEASE I CAN
BUILD A DESIGN AND TEST FOR THE
MUTATION.
IF I PUSH THE BUTTON NOW
ACADEMIC PRICES IT WILL TAKE
$12.5 MILLION AND 4 1/2 YEARS TO
PUT THAT UNDER A CLINICAL TRIAL
UNDER THE CURRENT AND REGULATORY
FRAMEWORK.
THE OLD WAY.
UNFORTUNATELY THERE ARE DOZENS
OF GENE MUTATIONS AND I DON'T
MEAN TO BE DRAMATIC BUT WE HAVE
A NEW PRODUCT AND THAT MEANS
WE'RE BACK TO SQUARE ONE.
THIS IS UNACCEPTABLE WE HAVE TO
GET TO A WORLD TO LEVERAGE EARLY
REGULATORY INFORMATION TO
THERAPEUTICS MUCH FASTER AND
MORE AFFORDABLY.
WE HAVE ENORMOUS MOMENTUM.
THE MOST IN MY YEARS IN GENE
THERAPY AND DOING GENE EDITING.
THE BIGGEST MOMENTUM AT THE
FEDERAL COMES FROM THE HEAD OF
THE CBER.
IN WASHINGTON HE SHOWED THE
SLIDES AND ARGUED FOR THE
MANUFACTURING OF THIS
THERAPEUTICS AND IF YOU HAVE AN
ORIGINATOR PRODUCT WITH THE GENE
INSERT AND TREAT A RELATED
DISEASE AND CHANGE THE INSERT
YOU SHOULD BE ABLE TO LEVERAGE
NON-CLINICAL INFORMATION FROM
ONE PRODUCT TO ANOTHER.
THAT'S EXCITING.
MORE EXCITING, THIS YEAR
DR. MARKS SAID THERE COULD BE A
BUT OF TRIALING AND GENE
EDITING.
A NEW DRUG APPLICATION AND THEN
BIOLOGIC SLICE APPLICATION FOR
DISEASE COULD CONTAIN MANY
EDITORS TO REPAIR DIFFERENT
MUTATIONS AND ALSO THE
MANUFACTURING PLATFORM.
THIS IS REMARKABLE AND ALSO
PITCH PERFECT CORRECT.
THAT IS THE WAY TO GET MEDICINES
FOR THE PATIENTS WITH MUTATION
WITH CMT IS TO BUCKETIZE THEM.
HOW DO YOU DO THAT?
FIRST, THEY PUT OUT GUIDANCE AND
THE PLATFORM FOR CRISPR AND GET
IT REVIEWED.
THAT IS WHAT WE'RE DOING AT THE
INNOVATIVE GENOMICS INSTITUTE
CONVERTING THIS TO A CIRCLE
WHERE THE GOAL IS TO ENGINEER
PLATFORMS AND I'LL EXPLAIN AND
THEN CLOSE ON THE ACTIONS.
THIS IS A PARTNERSHIP WITH
DANAHER.
THEY OWN ALL THE BITS AND PIECES
YOU NEED TO TAKE IT FROM
PARTICIPANT TO CLINICAL TRIAL
AND THIS IS A CROSS-FUNCTIONAL
TEAM' BEST IN CLASS TEAM I CALL
THE AVENGERS.
WHY AM I SHARING THIS WITH YOU?
WE'RE NOT TREATING IMMUNOLOGIC
DISORDERS WELL.
A KEY PART OF OUR EFFORT IS TO
SOLVE FOR THE PROGRAM.
CONSIDER A SETTING WHERE WE'LL
HAVE AN OPEN IND TO TREAT A
DEVASTATING ERROR OF THE IMMUNE
SYSTEM THAT AFFECTS NEWBORNS BY
GENE EDITING.
EVERY PATIENT BRINGS THEIR OWN
MUTATION.
YOU CAN HAVE A BEAUTIFUL IND AND
ALL THE SAFETY DATA BUT THE
NEWBORN MAY NOT HAVE THAT
MUTATION.
WHAT DO WITH DO?
WE NEED TO BE ABLE TO LEVERAGE
THAT IND AND TREAT THE CHILD
FAST AND AFFORDABLY AND THAT'S
WHAT WE'RE DOING.
FIRST, WE'LL BUILD ASSESSMENTS
AND USE THE SAME DELIVERY
PROCESS WHETHER IT'S MUTATION
ONE OR 57.
AND MOST IMPORTANTLY AND THIS IS
THE SECOND MOST IMPORTANT SLIDE
I'LL SHOW YOU TODAY, WE'LL BUILD
A BESPOKE GENE EDITOR.
YOU'LL SAY I'VE PROBABLY HAD TOO
MUCH CAFFEINE THIS MORNING.
NO, LET ME SHOW YOU HOW IT'S
DONE.
IF YOU WANT RESEARCH USED AND
RNA AND CRISPR IT WILL TAKE A
WEEK AND $100.
IF YOU WANT CGMP IT WILL TAKE A
YEAR AND A MILLION AND BY THIS
TIME THE KID COULD BE DEAD AT
LEAST EIGHT MONTHS.
WE'RE WORKING WITH DANAHER AND I
APPRECIATE THEIR PARTNERSHIP TO
ESTABLISH A MANUFACTURING
STANDARD AND PROCESS CALLED
PHASE-APPROPRIATE GMP.
ONE CAN MAKE A BESPOKE GENE
EDITOR FOR THIS CHILD OR MARY
WITH THIS DEVASTATING DISORDER
THAT CAUSES SEVERE CMT.
RAPIDLY, AFFORDABLY AND HOW DID
WE FIND THIS FOR $100.
I WANT TO SALUTE OUR PUBLIC
ACCESS TEAM FOR PUTTING OUT THE
REPORT.
I DO NOT WANT TO OVERSTEP MY
BOUNDARIES AND TALKING ABOUT
PHARMACY PLANS OR CMS BUT I WANT
TO SHOW THE TABLE.
THE GENETIC MEDICINES COST $2
MILLION, $3 MILLION, $4 MILLION
AND THE COMPANIES STILL CAN'T
FIGURE OUT HOW TO MAKE MONEY.
INNOVATION NEEDS TO HAPPEN FOR
MEDICINE TO BE $250,000 BEARABLE
BY THE HEALTH CARE SYSTEM BUT
YOU NEED TO BE ABLE TO MAKE THE
STUFF RAPIDLY AND AFFORDABLY AND
THAT'S WHAT WE'RE DOING IN
PARTNERSHIP WITH DANAHER.
BEFORE I GIVE TO THE LIST OF
ACTION ITEMS FIRST ONE IMPORTANT
LEARNING FROM THE REMARKABLE
WORK WHICH JUST CAME OUT IN
NATURE.
IF YOU THINK ABOUT HOW YOU TAKE
IN GIVEN MEDICINE FROM DISCOVERY
AND PRECLINICAL TO DEVELOPMENT
AND THEN MANUFACTURING, THERE
REALLY NEEDS TO BE FUNDAMENTAL
INNOVATION IN HOW THIS IS WOVEN
TOGETHER WITH A MUCH LARGER ROLE
FOR THE PUBLIC SECTOR.
BEFORE YOU SAY, FYODOR, INDUSTRY
SHOULD BE DOING THIS.
SADLY, MY COLLEAGUES IN INDUSTRY
ARE HAVING THE TOUGHEST TIME
EVER WHICH IS PARADOXICAL
BECAUSE THE TECHNOLOGY WORKS
WELL BUT THEY'RE HAVING LAYOFFS
AND DON'T WORK ON THE GENETIC
DISEASE BECAUSE IT'S NOT CLEAR
HOW TO COMMERCIALIZE FOR THESE
AND THEY TAKE TOO LONG TO
DEVELOP SO THE FOR-PROFIT SECTOR
DROPPED THEM.
I'M NOT EXAGGERATING.
THIS IS NOT HYPERBOLE AND
THERE'S A WAY TO CREATE
ORGANIZATIONS DEEPLY INTERWOVEN
WITH ACADEMIC INSTITUTIONS AND
UNIVERSITIES AND ULTIMATELY
PUBLIC BENEFIT CORPORATIONS
CONNECTED WITH MANUFACTURING
CAPABILITY TO BE AVAILABLE.
AND I SPOKE ABOUT CLINICAL TRACK
RECORD, IMPROVEMENT IN DELIVERY,
INNOVATIVE PUBLIC-PRIVATE
PARTNERSHIPS BUT AT THE END OF
THE DAY WHO IS GOING TO TREAT
MARY OR THE SEVERAL MILLION
PEOPLE LIKE HER IN THE WESTERN
WORLD ALONE WITH DEVASTATING
DISORDERS?
I HAVE A SPECIFIC.
AS YOU THINK OF GOING FROM A
RARE POINT MUTATION AFFECTING
ONE HUMAN BEING TO HUNTINGTON'S,
TOURETTE'S OR PAIN OR OTHERS THE
STEPS ARE FIRST WE MUST EXPAND
THE SPECTRUM OF NEUROLOGIC
DISORD
DISORDERS WE NEED DE RISKED
STREAMLINE AND A REGULATORY
PROGRAM.
WHO IS GOING TO DO ALL OF THAT?
THIS FOLKS IS THE ORIGINAL
DESIGN OF THE GREAT SEAL OF THE
UNITED STATES OF AMERICA 1782.
THIS IS THE CURRENT FORM AND I'M
SHOWING THIS BECAUSE THERE'S
ALSO A PLURBUS UNUM.
THE FEDERAL GOVERNMENT SHOULD
PLAY A BIGGER ROLE.
HERE'S WHAT I MEAN BY THAT.
LET ME GIVE YOU A CASE.
THE SOMATIC GENE EDITING
CONSORTIUM HAS A MEMORANDUM OF
CENTERS WITH EVALUATION IN
RESEARCH WHICH ALLOWS RAPID
FEEDBACK TO ENABLE NIMBLE
DEVELOPMENT.
THIS PROGRAM HAD A MAJOR
QUESTION FOR HOW TO DO A
SPECIFIC NON-CLINICAL THING.
WE DID NOT KNOW HOW TO DO THIS.
THE APPROACH SAVED THE
U.S. TAXPAYER THIS MUCH MONEY
BECAUSE INSTEAD OF DOING
SOMETHING A MILLION DOLLARS AN
EXPERIMENT WE'RE DOING IT AT
$10,000 AN EXPERIMENT.
THINKING ABOUT A DELIVERABLE IN
M.V.P. NEUROGENETIC DISEASE
CRISPR CURES COOKBOOK AND PAST
REGULATORY AND CLINICAL TRIAL.
THE SOONER IT HAPPENS THE SOONER
ONE CAN THINK OF PLATFORMIZING
CURATIVE APPROACHES TO THEM.
FURTHER, THERE'S A KNEAD TO
DISCOVER AND TEST BIOMARKERS FOR
EFFICACY IN NEUROGENETIC
DISEASE.
I DON'T THINK THE FOR-PROFIT
SECTOR WILL DO EITHER OF THOSE
TWO THINGS AND HUMBLY POSE TO
YOU I THINK THIS IS SOMETHING
THE FEDERAL GOVERNMENT
SPECIFICALLY THE NIH AND YOU CAN
GUESS WHY INSTITUTE COULD TAKE A
LEADERSHIP ROLE IN SUPPORTING
AND ENABLING.
THE PARTNERSHIP NEEDS TO BE
FORMALIZED AND EXPANDED.
I SPENT 16 YEARS IN INDUSTRY.
YOU CAN TAKE A LONG TIME TO FIND
OUT SOMETHING FROM THE AGENCY
AND SOMETIMES IT'S NOT ENTIRELY
CLEAR.
THAT'S THE WAY THE WORLD WORKS.
THIS IS A DIFFERENT MODE WHERE
BRANCHES OF THE GOVERNMENT NEEDS
TO BE EXTRAORDINARY AND THERE
NEEDS TO BE MORE.
THE SOMATICS OF GEN EDITING
CONCEPT NEED TO BE EXPANDED TO
WHAT IS CURRENTLY NOT FUNDED
BECAUSE THERE'S ONLY SO MUCH IN
EACH SPECIFIC BUCKETS.
THE TAUOPATHIES AND OTHERS ARE
TO BE STUDIED AND THERE NEEDS TO
BE A CRISPR THERAPEUTIC PROGRAM.
I MENTIONED OUR PROGRAM AGAIN
BUT THERE'S MANUFACTURERS WHO
MAKE CRITICAL COMPONENTS FOR
CRISPR AND NEED TO BE INTEGRATED
SO THE PIPELINE COULD EXIST.
TO BE CLEAR IT'S NOT A ZERO SUM
GAME FOR THE FOR-PROFIT SECTOR
BUT IT WOULD BE A RISING TIDE.
FINALLY, I THINK THIS COULD HAVE
THE GREAT SEAL OF THE UNITED
STATES.
THERE WAS A ROBUST FRAMEWORK FOR
COLLATING AND NIMBLE TO
INNOVATION, VIABLE PRODUCT.
WHERE THAT EXISTS WHEN PEOPLE
WHO TREAT THE NEXT MARY CAN JUST
CLICK AND DOWNLOAD THAT IT WILL
COMPLETELY CHANGE THE LANDSCAPE
OF HOW THIS HAPPENS.
I HAVE THE CMS LABEL AS MY LAST
FOR A REASON.
AS OTHERS ARGUE IN NATURE, THIS
NEEDS TO BE FORWARD AND
INTEGRATED INTO THE HEALTH CARE
ECONOMICS COMPONENT.
CRISPR IS A CHEMICAL SYNTHESIS.
WHEN AN ONDEMAND CRISPR EDITOR
CAN BE MADE AND DE RISKED FOR
$100,000 AND THIS WILL HAPPEN IT
WILL BE COVERED.
I'M HUMBLED FOR THE OPPORTUNITY
TO SHARE OUR THOUGHTS WITH YOU.
THANK YOU, DR. KOROSHETZ AND I
ALWAYS CLOSE WITH THE SAME SLIDE
BECAUSE I DON'T KNOW A BETTER
WAY TO EXPLAIN HOW TO BE AND
LIVE AND THIS IS POIGNANT IN THE
CONTEXT SHE SAID MERGE MERCY
WITH MIGHT AND MIGHT WITH RIGHT
AND I COULD NOT FIND A BETTER
WAY TO DESCRIBE THE WAY THE
FEDERAL GOVERNMENT CAN PLAY IN
EXPANDING THE ROLE OF CRISPR IN
GNK DISEASE.
-- GENETIC DISEASE.
THANK YOU VERY MUCH FOR YOUR
TIME.
>> THANK YOU.
WHILE OPEN FOR QUESTIONS BUT
FIRST I THINK DAN RONESTEEN IS
ON.
ANY COMMENTS YOU WANT TO MAKE?
>> THE ONLY THING TO ADD FOR US
IN THE GENETICS ESPECIALLY
COMMUNITY KNOWING THE WORK THE
IGI AND OTHER ORGANIZATIONS
ACROSS THE COUNTRY ARE DOING AND
NINDS INVESTIGATORS MOVING THIS
CLOSER TO THE TREATMENT OF
ESPECIALLY FIRST OF ALL THE
DEVASTATING EPILEPTIC
ENCEPHALOPATHIES IS EXCITING AND
AS YOU KNOW THE GENETICS AND
EPILEPSY WORKSHOP YOUR TEAM
ORGANIZED IN JUNE FELT LIKE A
WATERSHED MOMENT IN THE FIELD
BECAUSE GENETICS HAVE LED THE
ADVANCES OVER THE LAST 20 YEARS
AND NOW ARE ABLE TO DIAGNOSIS
DIAGNOSE AN AMAZING NUMBER OF
PATIENTS ESPECIALLY INFANTS AND
CHILDREN TO THE POINT WE KNOW
THE GENETIC ABNORMALITY AND
THERE'S TREATMENTS COMING ON
BOARD.
I APPRECIATE YOUR INVOLVEMENT.
THANK YOU.
>> THANK YOU FOR YOUR LEADERSHIP
AND FOR THE OPPORTUNITY TO SPEAK
TO THE EPILEPSY COMMUNITY.
>> THANKS, DAN.
THANKS FOR COMING BACK TO
COUNCIL AFTER WHAT MUST BE 15
YEARS.
>> YEAH.
>> VERY PROVOCATIVE TERM IN
TERMS OF THINKING THINGS FORWARD
AND HOW TO SCALE UP.
QUESTIONS?
>> I THINK IN SMALL MOLECULE
DEVELOPMENT THE CHALLENGES ARE
SIMILAR IN THE SENSE THE
DELIVERY AGAINST THE RIGHT
TISSUE THERE'S MOLECULAR
EFFICACY YOU WANT TO HIT THE
THINGS YOU THINK THE DRUG IS
DESIGN FOR WELL AND WORK IT AND
MINIMIZE OFF TARGET
POSSIBILITIES FOR THE DRUG.
IN THE DISCUSSION ABOUT I GUESS
FROM PETER MARKS' BUCKETING
APPROACH THINKING OF BECAUSE EF
THIS DETAILED CODING I DON'T
KNOW HOW MUCH YOU HAVE TO WORRY
ABOUT OFF TARGETING EFFECTS.
>> ASSESSING PHARM TALKS IS
COMPROMISED.
WHAT I MEAN IS GIVE ANY
PROFESSIONAL EDITOR SUCH AS
SOMEBODY AT THE IGI A GIVEN
MUTATION AND THE PIPELINE TO
SPIT OUT THE TOP EIGHT OPTIONS
HOW TO FIX IT IS AUTOMATED.
THE IN CELL PIPELINE TO
DETERMINE THE PHARMACOLOGY IS
TURN KEY AND IMPORTANTLY AS
SHOWN AND A RANGE OF OTHERS U
ULTIMUL
ULTIMIZING IS TURN KEY AND IT'S
A SYNTHESIS OF A NUCLEAR ACID
WHEN YOU HAVE IT AUTOMATED THE
DERISKING IN TERMS OF POTENCY
AND SPECIFICITY PLATFORMIZE AND
CASTS A LIGHT ON WHERE THE
BOTTLE NECK IS WHICH IS HOW TO
TREAT MORE PEOPLE WITH THE
PLATFORM.
I WANT TO BE CAREFUL.
THERE COULD BE OFF TARGETS WE
DON'T KNOW ABOUT OR MUTATIONS WE
CANNOT REPAIR BUT THANKFULLY AS
YOU KNOW WE HAVE TO GO TO THE
CLINIC TO FIND OUT HOW TO SAFELY
EDIT PEOPLE.
I'M NOT ARGUING FOR ANYTHING
BENIGN BUT THANK YOU FOR YOUR
QUESTION.
>> I UNDERSTAND THERE'S ONLY
CERTAIN STATES THAT GIVE
BENEFITS FOR PUBLIC BENEFIT
CORPORATIONS.
COULD YOU DIG INTO THAT A LITTLE
BIT AND HAVE YOU LOOKED INTO
THAT?
I WISH DR. CLEVELAND WERE HERE.
>> ANY STATE BY STATE
SUBSTANTIATION THAT COULD BE
VIABLE IF ULTIMATELY THAT
PROVIDES A PATH TO CLINICAL
TRIAL DATA TO BEGIN WITH AND
ULTIMATELY TO EVEN A LOCALIZED
SOLUTION AND WE COULD DO A
CONTROL ONCE WE KNOW WHAT THE
WORKING BITS ARE AND I REALIZE
IT'S A GENERIC QUESTION.
MY NAME IS ON THE WONDERFUL
PIECE BECAUSE I HAD TO SAY A FEW
THINGS ABOUT EDITING RATHER THAN
PVCs.
I'M EMBARRASSING MYSELF A LITTLE
BIT.
>> NO, NOT AT ALL.
THANK YOU.
THAT VERTISE WHAT WE'VE BEEN
FINDING AND I'LL DIG INTO THE
TOPIC A LITTLE BIT MORE.
MY SECOND QUESTION I HAVE TO
THANK YOU FOR POINTING OUT THE
ECONOMIC ISSUES THAT COME GENE
EDITING AND MAKING GENE EDITING
AVAILABLE ON THE SNA COMMUNITY
FOR SOME TIME ABOUT THE COST
RELATED TO ACCESS TO THAT GENE.
IT SPEAKS TO THE SECOND VALLEY
OF DEATH FOR THE NEW
TECHNOLOGIES COMING FORWARD IS
CLINICAL ADOPTION AND HOW DO
PEOPLE PAY FOR THAT.
IN YOUR PROPOSITION HOW DO YOU
INTEGRATE THE HEALTH ECONOMIC
STUDIES TO HELP ALLOW GETTING
OVER THE SECOND VALLEY OF DEATH
OF CLINICAL ADOPTION AND ACCESS
FOR THE COMMUNITIES THAT WE'RE
TARGETING KNOWING THEY'RE OUT
THERE.
>> I HAVE SPOKEN WITH PEOPLE WHO
THINK ABOUT THIS AND THE DUKE
MARGOLIS INSTITUTE INVITED ME TO
WORKSHOPS IN THAT SPACE AND I
GOT TO TALK TO PEOPLE ABOUT DO
THIS FOR A LIVING.
FIRST, THE COST OF GOODS SOLD OF
THE THERAPEUTIC CANNOT BE WHAT
IT IS RIGHT NOW.
ONE REASON THE COMPANIES CHARGE
THIS MUCH THEY'RE RECOUPING THE
COST OF SYNTHESIZING.
CRISPR IS A CHEMICAL SYNTHESIS
AND THE SCALABLE MANUFACTURE I
DESCRIBED COULD PUSH THE COST OF
THAT TO REALLY BELOW $100K.
I THINK IT'S IMPERATIVE WE NOT
TREAT GENETIC DISEASES BASED ON
INDIVIDUAL MUTATIONS.
WE CANNOT HAVE A SETTING WHERE
AN APPROVED DRUG IS A CRISPR
THAT FIXES THE MUTATION IN MARY.
IF WE GO THROUGH THAT PROCESSING
THE COST CAN DESTROY A COMPANY
EVEN THE SIZE OF LILY.
THE LICENSE AUTHORIZATION HAS TO
HAVE EMBEDDED IN IT THE ABILITY
TO MANUFACTURE AND ADMINISTER TO
A PATIENT AN INDIVIDUALIZED
CRISPR.
IN ALL OF MY CONVERSATIONS WITH
PEOPLE FROM CMS FROM
PUBLIC-PRIVATE INSURERS PEOPLE
WHO THINK ABOUT PLANS, BASICALLY
THE NUMBER FROM $100,000 TO
$200,000 PER PATIENT I'M TOLD IS
THE REALISTIC PRICE TAG WHERE IT
COULD BE SUSTAINABLE WHERE RIGHT
NOW IT'S $3 MILLION TO $4
MILLION AND WE'RE NOT ASKING FOR
A $50 CRISPR AND MY
UNDERSTANDING IS THE MECHANICS
COULD WORK OUT.
I HOPE I DID NOT OVERSTEP BY
SAYING THINGS I DON'T COMPLETELY
UNDERSTAND.
EICHLER HOW GOOD TO SEE YOU.
A FELLOW GENE EDITOR.
>> AS ALWAYS A TOUR DE FORCE.
SO MUCH TO LEARN FROM YOU.
I LOVE HOW YOU'RE TRYING TO
SCALE UP AND ACCELERATE THIS AND
BRING IT TO THE CLINIC.
FROM THE PERSPECTIVE OF A SIMPLE
CLINICIAN DOING GENE THERAPY I
SEE OTHER PERSPECTIVES AND
DIMENSIONS THAT ARE HOPEFULLY
SYNERGISTIC.
THE DELIVERY SHOULD NOT BE THE
OBSTACLE BUT STANDING IN THE WAY
OF SCALING UP BECAUSE DISEASE
CONTEXT MATTERS AND WHAT CELLS
WE WANT TO GET TO AND WHAT IS
OFFERED AND THE REACTIONS TO
VIRAL PARTICLES AND HAVEN'T
STANDARDIZED OUR ASSAYS AND
DON'T KNOW HOW TO INSERT OUR
GENESIS ACROSS TRIALS.
I WORRY A LITTLE BIT AS WE'RE
GOING TO CLINIC HOW CAN WE LEARN
SO WE DON'T HAVE SETBACKS LIKE
20 YEARS.
THERE'S ASOs AND VIRAL VECTOR
AND CRISPR.
IN MANY WAYS ASOs ARE PROOF OF
CONCEPT TARGETING SINGLE RNA
WORKS WHY NOT USE THAT TO
LEVERAGE AND CONVERT TO A CHRIS
PER.
HOW DO YOU THINK ABOUT THOSE
KINDS OF PAIRINGS AND CONTEXTUAL
THINKINGS.
>> SIMPLE CLINICIAN IS NOT THE
WAY TO DESCRIBE THEM.
IT'S ABOUT THE CLINICAL
EXPERTISE AND MASTERY.
ASOs TRACK FOR PATH AND CRISPR
FOLLOWS.
THE NIH FUNDED PROGRAM TO DO
GENE EDITING FOR SMA I THINK
THEY'LL DOSE MODEST AMOUNTS OF
THE EDITOR AND TAKE IT AND SEE.
I AGREE WE HAVE TO TAKE ALL THE
LEARNINGS FROM THE ASO COMMUNITY
AND BE A FAST FOLLOW WHERE THE
KEY ISSUES HAVE BEEN TO SOME
EXTENT ADDRESSED.
YOUR FIRST QUESTION ABOUT THE
REALM OF THE KNOWN AND UNKNOWN
DELIVERIES AND COMPLEXITY OF
DISEASE BIOLOGY, I THINK IT'S
OBJECTIVELY THE BIGGEST
OBSTACLE.
LET ME OFFER A WAY THE FIELD HAS
PROGRESSED THAT COULD BE A
CHARTED PATH.
I MENTIONED NANO PARTICLE
DELIVERY OF A BASE EDITOR TO
DISRUPT SPLICING FOR
CARDIOVASCULAR DISEASE.
THEY CHOSE TO USE WHAT EXISTED
AT THE THE TIME, ADENO
ASSOCIATED VIRUS.
FURTHERMORE, BASE EDITING DIDN'T
EXIST.
IT WAS INVENTED IN 2017.
THEY CHOSE TO USE A CONVENTIONAL
NUCLEASE.
THE WAY I'M THINKING ABOUT IT
AND THINK IT'S THE SEG IS A FIVE
YEAR PROGRAM AND NOT TWO-YEAR
PROGRAM.
THERE'S INCREMENTAL PROBLEMS OF
THE SPACES COULD LEVERAGE
PARALLEL TRACK ADVANCES ACROSS
ALL THE FACETS.
I'M ARGUING I AGREE WITH YOU
THERE'S SO MUCH WE DON'T KNOW
ABOUT HOW TO SAFELY DELIVER
LARGE AMOUNTS OF CRISPR TO LARGE
CHUNKS OF THE NERVOUS SYSTEM BUT
BUILDING FEDERALLY SUPPORTED
PROGRAMS THAT DO SO IN A
MEASURED WAY WE'LL BE ABLE TO
LOOK AT INNOVATION FROM AROUND
THE FIELD TO SAY LOOK WHAT WAS
JUST INVENTED AT STANFORD?
LIKE I DON'T WANT TO BE
POLLYANISH BUT IT COULD WORK.
>> STIMULATING.
THANK YOU FOR THAT.
BECAUSE SOME BASIS FOR BEING
WORRIED ABOUT ELEMENTS.
IT THE PLATFORMIZATION INVOLVED
THE PRIORITY SECTOR ENTITIES
COULD POTENTIALLY SLOW THINGS TO
SAY IF THAT COULD BE DISPERSED
AND SHARED AMONG ACADEMICS THE
WAVE WOULD BE FURTHER ADVANCED.
I'M INTERESTED TO HEAR YOUR
THOUGHTS HOW THAT COULD PLAY OUT
AND SECONDLY, SOME OF US HAVE
BEEN BRUISED BY CONFLICTS WITH
THE FDA AND HOPE WHAT YOU SAID
ABOUT PETER MARKS' WILLINGNESS
TO CONSIDER NEW MODELS WILL PAN
OUT BECAUSE WHAT YOU ARGUED IS
CONTINGENT OF A NEW WAY OF
THINKING IN THE FDA AND FOR
WALTER, THIS IS AN EXTRAORDINARY
SET OF DEVELOPMENTS.
THE NINDS IS A GREAT PLACE TO
START BUT THIS APPLIES ACROSS
SPECTRUM OF NIA MEDICINES.
>> THANK YOU FOR THE LEADERSHIP.
42 WAY TO THINK ABOUT THE KEY
COMPONENT OF INTELLECTUAL
PROPERTIES.
NEVER CONSIDER THIS AS A ZERO
SUM GAME.
WE'RE NOT TRYING TO PUT THE BIO
TECHS OUT OF BUSINESS.
THEY'RE PROPRIETARY SOLUTIONS
THAT CAN BE PLUG AND PLAYED IN
THIS PLATFORM SHOULD BE
PROTECTIVE.
I THINK THERE'S ENOUGH TO
KNOW-HOW IN THE FIELD TO HAVE
THE M.V.P. FOR CRISPR CURES
CONTAIN METHODS OUT THERE.
I'M SADDENED FOR MY COLLEAGUES
IN THE FOR PROFIT SECTOR BECAUSE
OF HOW MANY LAYOFFS THEY'VE HAD
TO DO.
A MAJOR GENE EDITING WENT $270
MILLION IN CASH TO NOBODY.
THEY ARE HAVING A TOUGH TIME
RIGHT NOW.
THAT WORK DOESN'T EXIST SO THEY
DON'T KNOW HOW TO MONETIZE THEIR
PLATFORMS.
I'M MODESTLY HOPEFUL WHEN WE DO
THIS THEY ARE WILL NOT SEE THIS
AS AN ENCROACHMENT OF THEIR IP
BUT ENABLEMENT TO APPROVE
MEDICINES.
I GOT SAD THINKING ABOUT MY
FRIENDS IN INDUSTRY AND FOR GET
YOUR SECOND QUESTION.
I'M SORRY.
>> THE LIKELIHOOD THE FDA WILL
RECOVER ITS WAY FORWARD?
>> IF I COULD PRAY FOR PETER
MARKS' LEADERSHIP I WOULD AND
THE DIRECTOR OF THERAPEUTICS AND
MANY FOLKS AT THE AGENCY, I
NEVER DREAMT THAT AT LEAST FOR
MY FOCUSSED FIELD OF CRISPR
CURES, CBAR LEADERSHIP WOULD
WELCOME INNOVATION AND PETER
MARKS SAID THIS YEAR AND NEXT
YEAR SHOULD BE THEIR POINT OF
OPENING THE SPACE TO NEW CURES
AND WE WANT TO GIVE SPECIFIC
PRACTICE.
WE SHOULD BE WALKING THE WALK AS
THEY TALK THE TALK.
I HAD MY DEALING WITH THE AGENCY
AND I THINK THERE'S MOMENTUM.
ONE STEP AT A TIME.
>> FABULOUS TALK AND VICKS.
6:
-- VISION.
THERE'S EDITING TECHNOLOGIES
WHERE YOU FIX THE BASE AND WHERE
THERE'S A REDUCTION IN GENE
FUNCTION YOU SURELY HAVE THOUGHT
ABOUT THE IMPACTS BIOLOGICALLY
AND HAVE YOU FACTORED IN THOSE
KINDS OF STUDIES IN TERMS OF HOW
THE SPEED WITH WHICH YOU'RE
PROGRESSING WILL TAKE INTO
ACCOUNT THE HETEROZYGOSITY
WITHIN STEM CELLS OR TISSUES HOW
THAT MIGHT IMPACT HEALTH AND
DOES THAT BECOME AN ISSUE AS YOU
THINK OF THE SPEED AND COST WITH
WHICH YOU CAN MOVE FORWARD
EFFECTIVELY IF THEY BECOME
ISSUES MOVING FORWARD?
>> YES WITH A CAPITAL Y.
MY FIRST PROGRAM TO DO GENOME
EDITING WAS HUNTINGTON'S AFTER
LEARNING ABOUT SELECTIVE TO
NON-SELECTIVE APPROACHES TO
SILENCE HDT AND FOR DOWN AND UP
REGULATION IF YOU THINK OF
ACTIVATING THE CHANNEL.
MY ADVICE IS WE SHOULD START IN
DISEASE INDICATIONS WHERE WE DO
NOT HAVE TO CLIMB THE MOUNTAIN
INITIALLY.
WE THUD -- SHOULD IS A READ OUT
AT A CELL, ORGANOID, NON-ANIMAL
MODEL AND NON-TARGET ENGAGEMENT
AND IN THOSE PLACES WHERE IT
BECOMES SECONDARY TO
ESTABLISHING THE MANUFACTURING
FRAMEWORK AND REGULATORY
FRAMEWORK AND PLATFORMIZATION.
WE HAVE THE IMPORTANT ISSUE OF
BI-DISTRIBUTION.
THE PROTEINS WE ENGINEER WHETHER
THEY'RE PROTEINS OR EPIGENOME
EDITORS THEY HAVE A GRADIENT
DISTRIBUTION OF THE TISSUE AND
AS THEY ARE DISTRIBUTE THE
PHARMACO DYNAMIC PROFILE CHANGES
BECAUSE OF DISTRIBUTION.
AS EVIDENCED BY THE RECENT
CLINICAL DATA WHERE THEY'RE
DOSING AAD FOR RHETT AND IT'S
ALMOST A DOSAGE RELEVANT GENE.
OTHER THAN A HUNTINGTON, MB2 IS
SURELY THE GENE.
CAUTIOUSLY AND WITH BENEFIT RISK
WE BASICALLY HAVE TO TRY.
I AGREE WITH YOU, MY PREFERENCE
LET'S JUST PREPARE A CAUSATIVE
MUTATION PRISTINELY AND THAT'S
THE STARTING POINT AND
UNFORTUNATELY THERE'S PLENTY OF
EXAMPLES LIKE THAT.
>> IN MANY CASES YOU HAVE TO
THINK ABOUT NOT JUST THE
DELIVERY OF THE TECHNOLOGY BUT
THE BIOLOGICAL TARGET AND I'M
COMING FROM THE DUCHENNE FIELD
WHERE WE HAD A MORTALITY WITH AN
EARLY CRISPR EMPHASIZING WHAT
YOU'RE DOING WITH THE UP AND
DOWN REGULATING AND WHAT DOES
THAT HAPPEN IN THE INDIVIDUAL
CIRCUMSTANCES VERSUS YEARS
PLATFORM MORE GENERALLY?
>> HOW MUCH TIME DO I HAVE TO
ADDRESS THIS POINT?
20 MINUTES?
JUST KIDDING.
THANK YOU, THANK YOU, THANK YOU.
WE HAVE HAD A TRAGEDY.
THE BROTHER OF A HUMAN BEING WHO
HAD MUSCULAR DYSTROPHY AND A
PERSON DIED WITHIN DAYS OF
DOSING.
SUBSEQUENT ANALYSIS SHOWED THE
PATIENT'S HEART WAS TOO FIBROTIC
AND IT OVERLOADED THE LATER BUT
AT THE END OF THE DAY EVERY
INDIVIDUAL IS THAT INDIVIDUAL OF
A CASE AND WE WILL NOT KNOWFUL
WE GO -- KNOW UNTIL WE TWO
FORWARD.
THE REASON I'M GRATEFUL FOR YOU
SAYING THIS IS WE LOOK AT THE
FIRST GENE THERAPY TRIAL AT THE
NIH IN 1989, I'M ARE NOT AWARE
OF A SINGLE ADVERSE EVENT THAT
WOULD HAVE BEEN PREDICTED FROM
NON-CLINICAL STUDIES.
CY
CYTEOKINE RELEASE AND THE CANCER
ON THE SICKLE CELL DISEASE
THERAPY TRIAL, NO SYSTEM TO
PREDICTS THAT.
TIM WU WILL BE THE FIRST TO SEE
THE ENCEPHALITITION IS --
ENCEPHALITIS DID IN THE SHOW
CERTAIN SIGNS AND I'M NOT A
PHYSICIAN I'M A SCIENTIST BUST
WE HAVE TO ACCEPT A CERTAIN
AMOUNT OF UNKNOWNS AS YOU KNOW
VERY WELL.
THIS IS WHY I THINK BENEFIT RISK
CONSIDERATIONS ARE PARAMOUNT AND
WHY THE ICG IS FOCUSSED ON
SEVERE DISEASES.
ULTIMATELY I'M CONVINCED THE SUM
TOTAL OF LEARNINGS WILL GIVE US
MORE OF A BIGGER PICTURE HERE
AND WHY REGULATORY LEARNINGS
COULD BE ACCEPTABLE SO WE DON'T
STEP ON THE SAME LAND MINES.
I AGREE.
>> I HAVE ONE QUESTION YOU DON'T
HAVE TO ANSWER BUT I'LL THROW
OUT, IN THE ECONOMIC MODEL TO
MAKE THESE AFFORDABLE ECONOMICS
CAN PLAY A ROLE WITH PARTNER AND
THAT MEANS THE NIH AND FDA BUT
THE UNIVERSITIES WOULD HAVE TO
MAKE A DEAL IN TERMS OF WHAT THE
COST IS GOING TO BE.
THEY WILL HAVE TO GIVE UP THE
CONTROL OF IP THEY CURRENTLY
HAVE WHICH IS USUALLY OVER
ESTIMATION OF THE VALUE HAVE YOU
HAD DISCUSSIONS ABOUT LIMITING
OR PUTTING TIES AROUND THE IP SO
THE FINAL THERAPY IS GOING TO BE
AFFORDABLE?
THAT WOULD HAVE TO START THERE.
THE UNIVERSITIES ARE NOT WILLING
TO DO THAT THEN IT'S A NO-GO
DOWN THE ROAD.
IF THEY DO IT, THEN THEY'LL
FORFEIT THE CHANCE TO MAKE A
LARGE AMOUNT OF MONEY SHOULD
THEY HIT A WINNER I'M WONDERING
WHAT YOU'RE THINKING THERE.
>> STAYING IN MY LINE HAVING
WORKED WITH IP PROFESSIONALS IN
THE PRIVATE AND PUBLIC SECTOR,
THE BUILDING I'M IN THERE'S
UNWITH PRINCIPLE.
WE MUST MAKE CRISPR SOLUTIONS
ACCESSIBLE AND AFFORDABLE TO
ALL.
THERE'S A DIFFERENCE ON FORMAL
ACTION THAT HOLD INTELLECTUAL
PROPERTIES RELEVANT TO THIS.
MY PROPOSAL IS THIS IS AN
EXCEPTIONAL OPPORTUNITY TO HAVE
THE DISCUSSIONS PROPER.
LET'S MAKE A CRISPR ON DEMAND
FOR EDITOR ON DEMAND AND IMAGINE
A SETTING AND I'LL GIVE A
SPECIFIC WHERE THERE'S A
COMPOSITION OF MATTER INVENTED
BY THE BROAD AND A SEPARATE GENE
EDITOR INVENTED BY U.C. BERKELEY
BUT THE BUCKET APPROVAL CONTAINS
BOTH.
WHAT DO WE DO WITH THAT?
WE'VE NEVER HAD THIS SITUATION
AND HOW DO WE HARMONIZE THE
INTERESTS OF INDIVIDUAL
INSTITUTIONS INCLUDING
UNIVERSITY OF CALIFORNIA HAVE IN
PROTECTING THEIR INVESTMENT WITH
IP FROM THE FORMIDABLE PUBLIC
GOOD.
WE'VE NEVER HAD A REASON TO ASK
THIS QUESTION WHERE THERE WOULD
BE A REASON TO DO THAT.
THE GOAL IS NOT TO HAVE THE
INSTITUTIONS GIVE UP IP FOR
LARGE SCALE COMMERCIAL PROJECTS,
SAY LILY JUST INVESTED IN A HUGE
GENETIC MEDICINE CENTER AND
PRESUMABLY ARE GOING TO DO
METABOLIC DISEASE.
THESE ARE HUGE MARKET.
WE'RE TALKING ABOUT DISEASES
WITH SEVERAL HUNDRED OR SEVERAL
THOUSANDS PATIENTS.
THESE ARE NOT HUGE MARKETS AND
WE'RE TALKING ONE AND DONE
CURATIVE THERAPIES TO BE
REIMBURSED AT 200K.
WE'RE HOPING TO CREATE AN
ENVIRONMENT WHERE IN THE BUCKET
OF CURES THERE COULD BE BUCKETS
OF COMPOSITIONS OF MATTER WHERE
UNIVERSITIES CAN CONTRIBUTE.
MAYBE I'M BEING OPTIMISTIC BUT
THANK YOU FOR THE QUESTION.
WE HAVE TO HAVE THE
CONVERSATION.
>> THANK YOU VERY MUCH.
WE'RE TRYING TO SELL TO
DR. BERTAGNOLLI TO SELL IT TO
CONGRESS AND WE'LL SEE WHAT
HAPPENS.
IF WE DON'T GRAB IT AND CONTROL
IT PRETTY SOON IT WILL BE OUT OF
OUR HARCHBDZ -- HANDS AND IT
WILL
AND THE COSTS COULD BE OUT OF
OUR HANDS AND I COMPLEMENT THE
GROUP FOR THINKING BEGINNING TO
END.
THAT'S WHAT WE NEED.
WE ALWAYS THINK ABOUT THE
BEGINNING AND HOW TO GET TO THE
NEXT STEP BUT YOU WANT TO KNOW
WHERE YOU'RE GOING WHEN YOU
START THE PROCESS OFF.
I APPRECIATE IT AND WE'LL
DEFINITELY BE IN TOUCH AND WE'LL
HAVE HURDLES TO GET OVER BUT
IT'S PRETTY CLEAR THIS IS THE
WAY TO GO.
>> THANK YOU.
>> AND BEHALF OF A HUGE TEAM OF
THE BUILDING BEHIND ME, THANK
YOU, THANK YOU, THANK YOU.
IT'S TRULY AN HONOR AS A PUBLIC
INSTITUTION WE STAND READY TO
SERVE.
THANK YOU.
>> THANK YOU.
>> THAT WAS FASCINATIFASCINATIN.
MOVING ALONG THE NEXT ITEM IS
OUR COUNCIL WORKING GROUP ON THE
NEURAL EXPOSOME PRIORITIES AND
I'M GOING TO PASS IT OVER TO
DAVE JETT AND PAM LEIN.
AND DAVE IS THE MUCH OUR OFFICE
OF TOXICOLOGY.
THISSING
THISSING OFF --
THIS OFFICE WAS LAUNCHED IN 2002
AND WANTED TO FOSTER MORE
SCIENCE ON THE NEURAL EXPOSOME.
WE THINK THIS WILL FOSTER MORE
SCIENCE.
REDUCE THE BURDEN OF ILLNESS.
THERE'S A STRATEGIC PLAN FOR THE
OFFICE THAT IS NEEDED TO ACHIEVE
THAT GOAL.
THE STRATEGIC PLAN WILL HELP
WITH THE ROAD MAP FOR OUTLINING
SHORT AND LONG-TERM GOALS AND WE
HAVE A WORKING GROUP ON THE TOP
PRIORITIES OF OUR STRATEGIC PLAN
AND THIS IS -- THE GROUP WILL
REPORT TO A NATIONAL ADVISORY
NINDS COUNCIL.
OUR OFFICE HAS THREE BRANCHS.
FOR NOW WE FOCUS ON THE NEURAL
EXPOSOME BRANCH AND WE WORK WITH
NIH AND OTHER AGENCIES.
SO THE NEURAL EXPOSOME.
ENVIRONMENTAL XHSHZ AFFECTING
THE GENOME WAS COINED AT
COVERING FACTORS THAT AFFECT
LEATH.
WE THINK STUDYING THESE RISK
FACTORS WILL UNCOVER THE CAUSES
OF A VARIETY OF HUMAN HEALTH
CONDITIONS INCLUDING THOSE WITH
OUR NINDS MISSION.
ONE TOX OFFICE WAS LAUNCHED TO
WORK ACROSS NIH AND WITH OTHER
AGENCIES TO SEE HOW THESE AFFECT
THE SYSTEM AND SUPPORT THE
RESEARCH WITHIN OUR MISSION.
AND AGAIN TO REDUCE THE BURTEN
ILLNESS.
IN A NEURON PAPER WE DESCRIBED
OUR THINKING OF WHAT WE THINK
LOOKS LIKE THE NEURAL EXPOSOME.
THERE ARE EXOGENOUS FACTORS LIKE
ENVIRONMENTAL TOXICANTS AND
LIGHT NOISE AND ENDOGENOUS LIKE
GENES AND GENETICS AND
MICROBIOME AND THINGS LIKE
STRESS AND SLEEP.
THE OFFICE HAS BEEN WORKING ON
THE NEURAL EXPOSOME FOR A COUPLE
YEARS NOW.
WE HAVE OUTREACHED TO THE PUBLIC
AND SOCIETY FOR NEUROSCIENCE AND
TOXICOLOGY WITH SCIENTIFIC
SOCIETY ON THE EXPOSOME AND HAVE
THE FORMATION OF A NIH WIDE
ENVIRONMENTAL SCIENCE WORKING
GROUP.
THE ONE TOX HAS PUBLISHED
FUNDING OPPORTUNITIES ACROSS
AREAS AND FUNDING ABOUT 20
PROJECTS AND WE HAVE TWO MORE
NOFOs OUT RIGHT NOW.
THIS WILL HAVE MANY MONTHS OF
DISCUSSION FOR LEADING THE
CHARGE AND THE CHARGE IS TO
DEVELOP RESEARCH PRIORITIES AND
ALSO TO PROVIDE RECOMMENDATIONS
FOR ENGAGING MORE NEURO
SCIENTISTS AND TOOLS AND
RESOURCES THAT WE'LL NEED.
WE HAD AN EXCELLENT AND STELLAR
WORKING GROUP.
WE ALSO HAVE OUR CHAIR AND A
LIST OF WELL KNOWN SCIENTISTS IN
EXPOSOME AND NEUROLOGICAL
FIELDS.
I THANK THEM SO MUCH AS WELL.
THIS WAS NOT A SIMPLE PROJECT
FOR THIS KIND OF THING.
AND I THINK AND THE STRATEGIC
PLAN COMMITTEE WILL DEVELOP THE
IMPLEMENTATION PLAN.
TODAY IT'S MY GREAT PLEASURE TO
INTRODUCE OUR CHAIR, DR. PAM
LEIN TO PROVIDE THE RESULTS OF
THE STRATEGIC PLANNING AND
PROFESSOR OF NEUROTOXICOLOGY.
CHAIR OF THE DEPARTMENT OF
MOLECULAR BIOSCIENCE AT U.C.
DAVIS SCHOOL OF MEDICINE.
HER LAB TACKLES MECHANISMS ON
PESTICIDES, TOXICITIES, AIR
POLLUTION AND NEURAL DEGENE
DEGENERATIVE DISEASE AND BETTER
TREATMENTS FOR CHEMICAL THREATS.
SHE HAS A BROAD KNOWLEDGE OF
BRAIN FUNCTION AND HOW
ENVIRONMENT MODIFIES DISEASE THE
EDITOR AND CHIEF OF THE PRIMARY
JOURNAL OF NEURAL TOXICOLOGY AND
I DON'T KNOW HOW MANY Ph.D.s
SHE'S HAD COME THROUGH HER LAB.
I'LL TURN IT OVER TO PAM.
>> THANK YOU, DAVID AND THANK
YOU FOR THE OPPORTUNITY TO
PRESENT THE WORK OF OUR
COMMITTEE AND WORKING GROUP.
I'D LIKE TO ACKNOWLEDGE I'M JUST
REPRESENTING THE OPINIONS OF A
VERY EXPERT GROUP AND MANY HAVE
EXPERTISE IN AREAS I DO NOT.
TO ECHO WHAT THE PREVIOUS
SPEAKER WAS SAYING THROUGHOUT
HIS PRESENTATION I'LL DO MY BEST
TO ANSWER QUESTIONS AT TO THE
END BUT THERE MAY BE AREAS WHERE
I'M NOT THE EXPERT AND I
APPRECIATE THOSE KEEPING ME
HONEST AND HELPING ME OUT WHEN
WE GET TO QUESTIONS.
WHAT IS THE EXPOSOME AND THIS IS
AN EVOLVING PART AND THIS WAS A
CONVERSATION THAT WAS ORGANIZED
BY DR. GARY MILLER A MEMBER OF
THE NEXT WORKING GROUP IN WHICH
THE GOAL OF THE CONFERENCE WAS
TO ADOPT A CONSENSUS DEFINITION
OF EXPOSOME AND EXPOSOMICS.
ACCORDING TO THIS PARTICULAR
CONVERSATION THE AGREED UPON
DEFINITION WAS THE INTEGRATED
COMPILATION AND THE OPERATIONAL
DEFINITION OF EXPOSOMICS STUDIES
THE COMPREHENSIVE AND CUMULATIVE
EFFECTS THAT AFFECT BIOLOGICAL
SYMPTOMS AND INTEGRATING DATA
FROM METHODOLOGIES AND DATA
STREAMS TO ENABLE DISCOVERY
BASED ANALYSIS OF INFLUENCES ON
HEALTH.
THAT'S A LOT.
THERE'S A MEET REPORT POSTED TO
THE CONFERENCE WEBSITE AVAILABLE
ON THE COLUMBIA UNIVERSITY
WEBSITE AND MANUSCRIPT UNDER
REVIEW AT SCIENCE AND WE EXPECT
IT TO BE ACCEPTED FOR
PUBLICATION ANY DAY NOW.
THE WORKING GROUP GRAPPLED WITH
THE EXPOSOME CHANGE AND WE'RE AT
THE BEGINNING STAGES OF THE
FIELD OF INQUIRINQUIRY.
IT'S DAUNTING TO THINK HOW WE
EXAMINE IMPACTS ON BRAIN HEALTH.
AND DETERMINE HOW THE INFLUENCES
PROMOTE NEUROLOGICAL DISEASE.
SO THE ONE THING WE HAVE TO STEP
BACK AND TELL OURSELVES IS YES,
WE'RE AT THE BEGINNING OF THE
FIELD AND IT SEEMS DAUNTING BUT
IT'S ALSO INCREDIBLY EXCITING
AND PROMISING.
WE NEED TO KEEP THAT INLINE AND
REMEMBER WE HAVE TO START
SOMEWHERE AND WHEN THE GENOME
STARTED 25 YEARS AGO IT SEEMED
DAUNTING.
BUT LOOK AT THE PROGRESS MADE.
SO WE WANT TO START BY
APPLAUDING THE NINDS FOR TAKING
THE INITIATIVE IN THE FIELD.
WE NEED FEDERAL INVESTMENT TO
MAKE THIS WORK AND CATALYZE
NOVEL ADVANCES IN THE FIELD AND
WHY SHOULD THE NINDS UNDERTAKE
THE INITIATIVE?
THERE'S A GENERAL SCIENTIFIC
CONSENSUS THAT MANY IF NOT MOST
NEUROLOGICAL DISEASES AND
DISORDERS ARE NOT DUE SOLELY TO
GENETIC CAUSES BUT FROM COMPLEX
INTERACTIONS BETWEEN GENETIC
SUSCEPTIBILITIES AND
ENVIRONMENTAL EXPOSURES AND RISK
FACTORS.
WE KNOW THIS BECAUSE MORE THAN
25 YEARS OF SIGNIFICANT
INVESTMENT IN RESEARCH HAS
IDENTIFIED THE CAUSE OF ONLY A
FEW NEUROLOGICAL DISEASES AND
DISORDERS AND REVEALED MANY
INCLUDING SOME OF THE MOST
COMMON DISABLING AND
NEUROLOGICAL DISEASES SUCH AS
STROKE, ALZHEIMER'S, PARKINSON'S
AND PAIN ARE AMONG THE LEAST
HERITABLE OF THE DISORDERS.
INVESTING IN RESEARCH AND
UNDERSTANDING ENVIRONMENTAL
FACTORS THAT INFLUENCE SYSTEM
HEALTH OFFER AN EXCITING
POSSIBILITY OF THE DECREASE IN
THE BURDEN OF NEUROLOGICAL
DISEASE BY CONTROLLING
EXPOSURES.
WE HEARD A BEAUTIFUL AND
EXCITING TALK IN GENE EDITING
THAT IS NOT READY FOR PRIME TIME
AND WILL TAKE INVESTMENT AND
IT'S AN EASIER ENVIRONMENT TO
CHANGE THAN OUR GENETICS BUT TO
LOOK AT PUBLIC HEALTH
STRATEGIES, THEIR FOCUSSED ON
EXPOSURE CONTROL WE NEED TO
UNDERSTAND WHAT ARE THE
ENVIRONMENTAL FACTORS THAT
INFLUENCE NEUROLOGICAL DISEASE
AND PATHOLOGY AND UNDERSTAND
MORE IMPORTANTLY THE MECHANISMS
BY WHICH THEY'RE DOING SO.
AND THERE'S DISCUSSIONS IN TERMS
OF RECOMMENDATIONS BY NINDS
WOULD BE INFORMED IF THERE'S
CRITICAL QUESTIONS TO ADDRESS
UNDERSTANDING THE EXPOSOME.
THESE ARE SUMMARIZED IN THE
SLIDE.
WHICH ENVIRONMENTAL FACTORS
INCREASE RISK OF NEURO LOGICAL
DISEASE?
HUMANS ARE EXPOSED TO A NUMBER
OF ENVIRONMENTAL EXPOSURES AND
ENDOGENOUS FACTORS.
WHICH ARE IMPORTANT DRIVERS IN
THE CONTEXT OF NEUROLOGICAL
DISEASE?
WHICH INCREASE RESILIENCY?
WE NEED TO THINK OF A
TWO-PRONGED ATTACK NOT ONLY
UNDERSTANDING WHAT'S PROMOTING
DISEASE AND WHAT IN OUR EXPOSOME
IS PROMOTING NEURO LOGICAL
RESILIENCY AND ARE THERE
SPECIFIC EXPOSOMES AND THIS IS
IS IMPORTANT TO IDENTIFY THE
EXISTENCE OF SUCH DISEASE AND
DISORDER SPECIFIC EXPOSOMES.
ANOTHER CRITICAL QUESTION MANY
EXOGENOUS AND ENDOGENOUS FACTORS
ARE ASSOCIATE WITH NEUROLOGICAL
DISEASES.
AIR POLLUTION IS A STRONGLY
ASSOCIATED RISK FACTOR FOR
STROKE, A.D., P.D.
WHAT DETERMINES IF AN INDIVIDUAL
EXPERIENCES AN INCREASED RISK
VERSUS EPILEPSY?
WHAT ARE THE CONTRIBUTIONS OF AN
INDIVIDUAL'S GENETIC SUBSTRATE
AND THEIR UNIQUE EXPOSOMIC
PROFILES IN RESPONSE TO
STRESSORS?
THIRD QUESTION, HOW DO
ENVIRONMENTAL FACTORS INTERACT
WITH EACH OTHER AND WITH THE
GENOME TO INFLUENCE NEUROLOGICAL
DISEASE RISK.
ARE SPECIFIC LIFE STAGES UNI
UNIQUELY SENSITIVE TO
ENVIRONMENTAL FACTOR AND WHAT IS
THE CUMULATIVE RISK OF BRAIN
HEALTH TO EXPOSURES TO MULTIPLE
FACTORS AND HOW DO EXPOSURES
EARLIER IN LIFE INFLUENCE
DISEASE RISK LATER IN LIFE?
IF WE CAN MAKE ADVANCES IN ANY
OR ALL THESE QUESTIONS WE'D BE
IN BETTER SHAPE FOR USING THE
KNOWLEDGE TO IMPROVE BRAIN
HEALTH AND INCREASE NEUROLOGICAL
RESILIENCY.
THE NEXT IDENTIFIED SIX PRIORITY
AREAS TO GUIDE INVESTMENT IN
EXPOSOME WORK.
THE FIRST IS IDENTIFYING
EXPOSOMIC FACTORS THAT INFLUENCE
NEUROLOGICAL HEALTH AND EVALUATE
HOW THEY ARE RESULT IN HAME AND
LOOK AT POPULATION BASED
RESEARCH STUDIES.
CREATE A MULTI-DISCIPLINARY POOL
OF INVESTIGATORS TO ASSESS THE
NEURAL EXPOSOME AND DEVELOP
RESOURCES AND TOOLS TO ASSESS
THE NEURAL EXPOSOME AND FOSTER
COLLABORATION AND PARTNERSHIPS
TO ACCELERATE AND AMPLIFY
RESEARCH.
FIRST, IN TERMS OF IDENTIFIED
EXPOSOMIC FACTORS THAT INFLUENCE
NEUROLOGICAL HEALTH.
THE NEXT WORKING GROUP
IDENTIFIED SEVERAL DATA CAPS
THAT CUT ACROSS DISCIPLINES AND
IMPLEMENTATION SCIENCE AND
PUBLIC HEALTH.
THESE INCLUDED THE
IDENTIFICATION OF SPECIFIC
ENVIRONMENTAL FACTORS THAT
INFLUENCE BRAIN HEALTH AND THE
CONTEXT THAT MODIFIES
NEUROLOGICAL OUTCOMES ASSOCIATED
WITH THESE EXPOSURES.
BY CONTEXT WE'RE REFERRING TO
THINGS LIKE LIFE STAGES,
CO-EXPOSURES, GENETIC MODIFIERS,
DISEASE MODIFIERS, ETCETERA.
ANOTHER CRITICAL DATA GAP WAS
THE NEED TO SHIFT TOWARDS
RESEARCH THAT ASSESS THE COM
COMPOSATE EFFECT AND IT'S
FOCUSSED ON A SINGLE EXPOSURE OR
OUTCOME AND HAVE TO SHIFT TO
MORE INTEGRATIVE STUDIES.
THIS IS NECESSARY TO DEVELOP AN
UNDERSTANDING HOW THE MULTIPLE
FACTOR INTERACTING TO INFLUENCE
BRAIN HEALTH AND HOW THEY
ACCUMULATE THE LIFE SPAN.
AND THE OTHER FACTOR IS
UNDERSTANDING THE IMPACT ON
BRAIN HEALTH.
ONE OF THE BIGGEST CHALLENGES
FACING OUR SPECIES IS CLIMATE
CHANGE AFFECTING EVERY ASPECT OF
OUR LIVES.
SOCIAL, HEALTH WISE, FOOD
SECURITY, HOUSING SECURITY,
ETCETERA.
HAVING A MAJOR IMPACT ON MENTAL
HEALTH.
HOW IS IT AFFECTING BRAIN HEALTH
AND NEUROLOGICAL RESILIENCY?
HOW DO WE USE THAT KNOWLEDGE TO
PROTECT OURSELVES AS A SPECIES
AS WE BEGIN TO DEAL WITH THE
IMPACTS OF CLIMATE CHANGE?
AND RESEARCH TO PROMOTE BRAIN
HEALTH EQUITY.
I KNOW NINDS HAS BEEN ENGAGED IN
A LOT OF RESEARCH, FUNDING
RESEARCH TO UNDERSTAND HEALTH
DISPARITIES.
THERE'S MORE REALIZATION THE
DISPARITIES ARISE AND NOT ONLY
THE SOCIO ECONOMIC SETTING BUT
THE ENVIRONMENTAL SETTINGS IN
WHICH THESE COMMUNITIES ARE
LIVING.
BEING ABLE TO LEVERAGE THE
RESEARCH IN THE AREA TO REALLY
THEN PROMOTE BRAIN HEALTH EQUITY
WOULD BE A HUGE STEP FORWARD.
AND THE NINDS HAS TRASHLDITIONA
FUNDED RESEARCH THAT SHOULD BE
LEVERAGED ON THE EXPOSOME WITH
IDENTIFYING NOVEL MITIGATION
TARGETS AND PRIORITY AREAS
INCLUDE THE UNDERPINNINGS OF
RESILIENCY ACROSS THE LIFE
COURSE AND ELUCIDATING HOW THEY
PROMOTE OR UNDERMINE RESILIENCE.
SECOND IS DEVELOPING HOW ADVERSE
FACTORS PROMOTE NEUROLOGICAL
DYSFUNCTION AND HOW THEY
INTERACT TO PROMOTE NEUROLOGICAL
DISEASE.
AND TO IDENTIFY THE MECHANISMS
BY WHICH DISEASE AND OTHER ORGAN
SYSTEMS PARTICULARLY
CARDIOVASCULAR, PULMONARY AND
EPITHELIAL SYSTEMS IMPACT
NEUROLOGICAL DISEASES.
THE EXPOSOME IMPACT THE ORGAN
SYSTEMS AND GIVEN THE INCREASE
CROSS TALK BETWEEN THE BRAIN AND
PERIPHERAL ORGAN SYSTEMS IN THE
CONTEXT OF BRAIN HEALTH IS
IMPORTANT TO UNDERSTAND HOW THE
EXPOSOME INTEGRATES ACROSS THE
ORGAN SYSTEMS.
THE THIRD PRIORITY AREA IS TO
EVALUATE EXPOSOMIC FACTORS IN
POPULATION STUDIES.
PUBLIC HEALTH, CLINICAL AND
INTERVENTIONAL RESEARCH STUDIES
ARE DESPERATELY NEEDED TO
IDENTIFY NEURAL EXPOSOME RISK
FACTORS AND NEED TO USE THE
KNOWLEDGE TO TEST STRATEGIES TO
MITIGATE SUCH RISKS.
CAN WE CHANGE THE COURSE OF
NEUROLOGICAL DISEASE MITIGATING
RISK FACTORS?
ACTION ITEMS INCLUDE IDENTIFYING
ENVIRONMENTAL RISK FACTORS THAT
PROMOTE NEUROLOGICAL DISEASES
AND COHORT TO ELUCIDATE
INTERACTIONS OF RELEVANCE TO
BRAIN HEALTH.
SECONDLY TO ASSESS THE IMPACT ON
NEUROLOGICAL HEALTH OF POLICY
AND COMMUNITY INTERVENTIONS THAT
MODIFY EXPOSOMIC FACTORS.
FOR EXAMPLE THERE'S NATURAL
EXPERIMENTS ONGOING ALL THE TIME
AND NINDS WOULD BE IN A GOOD
POSITION TO LEVERAGE THOSE.
SOME EXPERIMENTS INCLUDE CHANGES
IN PUBLIC POLICY FOR EXAMPLE WE
SAW IN MICHIGAN A HUGE PUBLIC
POLICY SHIFT REGARDING THE USE
OF LEADED PIPES TO DEVELOP
WATER.
WE NEED TO UNDERSTAND HOW MITT
GETTING LEAD EXPOSURE CHANGES
THE TRAJECTORY OR LOOK AT
GEOGRAPHICAL SETTINGS.
IT WOULD BE AWESOME TO HAVE
POLICIES AND PROGRAMS IN PLACE
TO LEVERAGE EXPERIMENTS AND
UNDERSTAND HOW MITIGATING
EXPOSURES CHANGES NEURO LOGICAL
DISEASE.
THIRD IS DEVELOP TEST STRATEGIES
TO MITIGATE EXPOSURES INCLUDING
STUDIES TO ASSESS AND EXPAND
RESILIENCE.
THE FOURTH PRIORITY AREA WAS TO
CREATE A POOL OF INVESTIGATORS
TO SATISFACTION THE NEURAL
EXPOSOME.
THE TRANS DISCIPLINARY NATURE OF
EXPOSOME RESEARCH REQUIRES
SUPPORTING AND BUILDING HIGHLY
SKILLED MULTI-DISCIPLINARY TEAMS
OF CLINICAL NEUROLOGISTS AND
TOXICOLOGISTS, BIO
INFORMATICIANS AND SUPPORT FOR
TRAINING AND EDUCATION AND
MULTI-DISCIPLINARY EXPOSOMICS TO
RESEARCHERS OF ALL CAREER
STAGES.
FEW FUNDED NOW HAVE THIS LEVEL
OF EXPERTISE TO LET US TRAIN TO
ADJUST.
SUPPORT THE INTEGRATION MUCH
EXPOSOMIC EXPERTISE TO ONGOING
HUMAN COHORT STUDIES.
PROVIDER SUPPLEMENTAL FUNDING TO
ADD EXPERTISE TO THE PROJECT.
ENSURE REVIEW PANELS.
HAVE THE APPROPRIATE EXPERTISE
TO EVALUATE SCOMBOES OME
FOCUSSED RESEARCH INCLUDING
EXPOSOME WIDE ASSOCIATION
STUDIES AND FOR NINDS TO
COMMUNICATE THE IMPORTANCE OF
THE RESEARCH.
A STRAIGHTFORWARD STEP WOULD BE
UPDATE THE WEBSITE AND
COMMUNICATION MATERIALS
INCLUDING MISSION STATEMENT TO
CONVEY THE COMMITMENT TO
UNDERSTANDING THE INFLUENCE OF
THE ENVIRONMENT OF BRAIN HEALTH.
EXPOSOMICS DIDN'T APPEAR IN THE
MISSION STATEMENT OR WEBSITE.
TO EDUCATE THE PUBLIC, POLICY
MAKERS AND DISEASE COMMUNITIES
AND JOURNALISTS IN THE ROLE THE
ENVIRONMENT PLAYS IN BRAIN
RESEARCH.
THE FIFTH WAS TO DEVELOP TOOLS
TO DEVELOP THE EXPOSOME.
AND THE EXPOSE I AM RESEARCH
WILL REQUIRE INTERDISCIPLINARY
APPROACHES ACROSS ALL BIOLOGICAL
LEVELS.
IT WILL BE CRITICAL FOR NINDS TO
INVEST IN THE DEVELOPMENT OF
RESOURCES AND TOOLS.
THIS IS A GOAL THAT PRETTY MUCH
IS LIKELY TO BE REALIZED BY
LEVERAGING ONGOING EFFORTS.
THERE'S CURRENTLY A LARGE NUMBER
OF EFFORTS TO DEVELOP THE
RESOURCES AND TOOLS AND NOW
INCREASING EFFORTS TO INTEGRATE
EFFORTS FOR A HARMONIZED
APPROACH AND DATA CAN BE SHARED
ACROSS PLATFORMS.
THERE'S AREAS WHERE TOOL
DEVELOPMENT CAN BENEFIT FROM
DATA REPOSITORIES AND
INFRASTRUCTURE AND SYSTEMS
INVOLVEMENT.
IN REGARD TO BIO AND DATA
REPOSITORIES NEUTRAL LIFE IS
DIFFICULT TO RECONSTRUCT IN
ADULT STUDIES.
WE NEED INFRASTRUCTURE TO ENABLE
THE DATA COLLECTION AND STORAGE
OF EXPOSOME DATA TO EXISTING
PHENOTYPE REPOSITORIES.
POLICIES TO ASSIST ALONG THESE
LINES WOULD BE HELPFUL.
WITH RESPECT TO INFRASTRUCTURE
THERE'S A REAL NEED TO INVEST IN
THE RESEARCH NEEDED TO
UNDERSTAND WHAT IS NEEDED TO
PROVIDE TECHNOLOGY AND
METHODOLOGICAL SUPPORT FOR
RESEARCH.
NOT SAYING NINDS NEEDS TO INVEST
IN BUILDING INFRASTRUCTURE BUT
IT WOULD BE GREAT IF IT COULD
INVESTMENT IN RESEARCH TO
PROVIDE INSTRUCTIONS MODELS AND
GUIDELINES FOR HOW WE DO AS A
LARGE COMMUNITY DEVELOP THE
INFRASTRUCTURE.
ANOTHER RECOMMENDATION IS TO
CONVENE AND FUND A WORKING GROUP
TO FOSTER DATA ACCESS AND
ADDRESS CULTURAL ISSUES THAT
IMPEDE DATA SHARING.
A HURDLE TO ADVANCING EXPOSOME
RESEARCH IS ACCESSING DIVERSE
DATA STREAMS AND INTEGRATE THEM
TO STUDIES.
NINDS HELP POUWOULD BE APPRECIA
AND WITH MODEL SYSTEMS THERE'S A
NEW APPROACH METHODOLOGY FUNDED
BY OTHER RESEARCH INSTITUTES
ALREADY.
THESE INCLUDE INSILICO AND
ORGANISM MODELS TO SCREEN
ENVIRONMENTAL STRESSORS TO ARE
THOSE THAT CONVERGE MECHANISTIC
PATHWAYS WE KNOW ARE IMPORTANT
IN THE PATHOGENESIS OF
NEUROLOGICAL DISEASE.
A SECOND MODEL SYSTEM THAT IS
RIPE FOR BEING LEVERAGED WOULD
BE NON-HUMAN ANIMAL DISEASES
COMPARABLE TO HUMAN NEURO
LOGICAL DISEASES.
FOR EXAMPLE, DOGS DEVELOP
EPILEPSY THATTING.
HE -- THAT ARE MECHANISTICALLY
SIMILAR AND UNDERSTANDING HOW
THE EXPOSOME INFLUENCES EPILEPSY
FOR DISEASE TREATMENT WOULD BE
FRUITFUL.
AND CAN LOOK AT DISEASE
INCIDENTS AND DISEASE
PROGRESSION.
THE LAST PRIORITY AREA FOSTER
COLLABORATION AND PARTNERSHIPS
TO ACCELERATE AND AMPLIFY
EFFORTS IN THE NEURAL EXPOSOME
IT'S CLEAR TO THE NEXT WORKING
GROUP THE TRANS-NIH PARTNERSHIPS
ARE NEEDED TO REALIZE THE
POTENTIAL POSITIVE IMPACT OF
NEURAL EXPOSOMICS ON HUMAN
HEALTH AND NINDS SHOULD EXPAND
TO INCLUDE MEASURES OF
NEUROLOGICAL HEALTH AND
INCENTIVIZE STUDIES OF BRAIN
HEALTH AND ADD EXPOSOMIC
MEASURES.
SEEK INPUT FROM INSTITUTES AND
CENTERS AND OTHER AGENCIES THAT
HAVE EXTENSIVE EXPERIENCE
ALREADY BUILDING INFRASTRUCTURE
AND TOOLS FOR COLLECTING,
MANAGING DATA AND LEVERAGING THE
INSTITUTE AND COLLABORATE WITH
PROGRAM TO INCENTIVIZE RESEARCH
ON THE ROLE OF ENVIRONMENTAL
EXPOSURE AND EXPRESSION OF
NEUROLOGICAL DISEASE AND SET
STANDARDS FOR ENVIRONMENTAL
QUALITY AND ENGAGE IN EFFORTS TO
ESTABLISH A NATIONAL OR GLOBAL
TOXICANT REGISTRY WITH EXPOSURE
DATA, FOR EXAMPLE, AIR POLLUTION
AND PESTICIDE EXPOSURES, NANO
PLASTICS MAPPED TO SPECIFIC
UNITS AND A NATIONAL GLOBAL
MONITORING PROGRAM.
WITH THAT I'M STOP AND TAKE ANY
QUESTIONS.
I WAS TOLD TO KEEP IT SHORT TO
HAVE ROOM FOR QUESTIONS SO HAPPY
TO FIELD QUESTIONS.
DAVE FEEL FREE TO ADD ON IF YOU
HAVE ANYTHING TO ADD.
I WELCOME THAT AS WELL.
>> IF EVERYBODY COULD STAND UP
AND STRETCH FOR A SECOND AND
THEN I'D BE WILLING TO GIVE AN
EXAMPLE OF WHAT COULD BE.
>> IF YOU WANT TO SABOTAGE A
PRESENTATION PUT IT AT 2:30 IN
THE RAFT NOON.
-- AFTERNOON.
>> YOU DID A GREAT JOB OF FLINT
OF THE KINDS OF THINGS WE'RE
TRAUKING ABOUT.
I WANTED TO GIVE AN EXAMPLE, I
JUST E-MAILED SINCE I SAW THE
RELEASE OF A NOFO FOR
ALZHEIMER'S DISEASE AND
EXPOSOMICS I E-MAILED EXPERTS IN
THE FIELD THAT COULD TO A STUDY
IN LOS ANGELES AND RISK FOR
ALZHEIMER'S DISEASE BUT IT WOULD
ALSO BE GOOD FOR STROKE OR OTHER
THINGS TO ADD OTHER ASSESSMENTS
INTO THIS AND NOT JUST
ALZHEIMER'S DISEASE RISK.
THE THINGS WE COULD COLLECT IN
LOS ANGELES WOULD HAVE TO BE
WORKING WITH THE COMMUNITY.
WE'D WANT TO KNOW WHAT HAIR
PRODUCTS ARE YOU USING.
DO YOU LIVE NEAR A FIRE STATION.
WHAT ARE THE STRESSORS YOU'RE
UNDERGOING, HOW MUCH TRAFFIC DO
YOU DRIVE IN EVERY DAY.
ALL THESE THINGS COME TOGETHER.
TO INFLUENCE THINGS LIKE
ALZHEIMER'S OR STROKE RISK.
NOW THAT WE HAVE THE TOOLS TO
ASSESS A BUNCH OF THINGS AND
HAVE THE DATABASES TO HANDLE THE
INFORMATION AND I THINK WE'RE
WELL POISED TO START THESE
INVESTIGATIONS THAN NEUROSCIENCE
HAS TAKEN US IN THE PAST.
AND LOOKING AT STUDIES ACROSS
DISCIPLINARY WORK THIS IS A
COMPREHENSIVE SET OF GUIDELINES.
THERE'S A SITUATION WHERE THERE
MAY BE MANY PROGRAMS COLLECTING
DATA RELATED TO OTHER ORGAN
SYSTEMS.
THEY HADN'T THOUGHT ABOUT THE
BRAIN.
THEY MAY BE COST EFFECTIVE.
WHAT ARE WE DOING IN THAT SPACE.
HOW IMPORTANT IS IT THAT THIS
STILL EXISTS?
>> THAT MIGHT BE A QUESTION FOR
DAVID TO ANSWER.
>> THE NEURAL BIO BANK IS THE
BIGGEST INVESTMENT AND TISSUE
SAMPLES WITH CLINICAL DATA
THERE'S SAMPLE ON LOTS OF PEOPLE
THERE'S OVER 10,000 PEOPLE WITH
WHOLE GENOME SEQUENCING,
PROTEOMICS.
THE BIG QUESTION IS WHAT ARE YOU
GOING TO MEASURE AND THAT HELD
THE FIELD BACK.
ANYBODY WITH A CLEVER IDEA THAT
CERTAIN EXPOSURES MAY CAUSE X
AND MAY CHANGE Y THAT'S WHAT A
CLEVER PERSON HAS TO SORT OUT.
WE HAVE PUT TOGETHER BIO
REPOSITORIES TO TEST THEORY.
THE PROBLEM IS WE CAN'T DO THAT
FOR EVERY DISEASE WE HAVE.
WE'RE KIND OF LIMITED THERE.
THERE'S A U.K. BIO BANK FOR THE
WORK WHERE THE WHOLE COUNTRY
COLLECTED SAMPLES AND CLINICAL
DATA ON 100,000 PEOPLE.
ALL OF US, WHICH IS WHAT NIH'S
PROGRAM IS TRYING TO GET TO A
MILLION PEOPLE WITH BIO SAMPLES
AND CLINICAL DATA WHO HAS HOPED
TO BE SOMETHING LIKE THAT IN THE
POPULATION SPACE, THEY GOT HIT
WITH THE SAME BUDGET CUT THE
BRAIN INITIATIVE DID.
THEY HAD ISSUES AS WELL.
PAM MENTIONED LOOKING AT A
CHANGE IN THE ENVIRONMENT AND
LOOKING FOR CHANGES.
THERE MAY BE HOT POCKETS WITH
EXPOSURES AND MAY MNOT BE IN THS
COUNTRY BUT TAKING ADVANTAGE OF
THE HIGH EXPOSURES VARIATION
AROUND THE WORLD TO SEE IF YOU
CAN SEE HOT SPOTS FOR CERTAIN
CONDITIONS BEING ASSOCIATED AND
THE OTHER PROBLEM IS YOU TALK
ABOUT EXPOSOME AFFECTING NEURAL
LOGIC HEALTH.
IF IT'S A SINGLE HIGH EXPOSURE
EVENT LIKELY THAT'S TRACTABLE.
IT'S NOT EASY BUT TRACKABLE.
IF IT'S A LOW EXPOSURE OF 50
YEARS THEN IT GETS HARD.
WE NEED ANOTHER INTEGRAL OF THE
EXPOSOME TO HAMMER THOSE OUT.
THOSE ARE GREAT POINTS.
I THINK THERE'S A COUPLE THINGS
EXCITING THAT ARE BEING
DEVELOPED.
ONE IS HIGH RESOLUTION IN
GEOSPATIAL MAPPING.
THERE'S NOW THE ABILITY TO
LEVERAGE HISTORIC DATABASES
WHERE FOR EXAMPLE NOAA HAVE
MAPPED PESTICIDES OR POLLUTION
AND WE CAN GO BACK 50 YEARS AND
MAP THEM PRECISELY TO SPECIFIC
GEOGRAPHIC LOCATIONS.
NOW WE HAVE CENSUS DATA TO SAY
WHO LIVED THERE WHEN AND THAT IS
A WAY TO COMPILE A LIFE TIME
EXPOSURE TO A LOT.
WE ALSO NOW HAVE FASCINATING
EMERGING TECHNOLOGIES WHERE WE
CAN LOOK AT LIFE TIME EXPOSURE
INTEGRATED IN TOOTH ENAMEL.
PEOPLE CAN ERODE LAYERS OF THE
TEETH AND LOOK AT AN UNBIASSED
MANNER A NUMBER OF CHEMICALS TO
SEE WHAT YOU WERE EXPOSED TO AT
THAT TIME IN YOUR LIFE.
THERE'S A LOT OF THAT GOING ON
IN THE CONTEXT OF CHILDHOOD
DISEASES TAKING SHED BABY TEETH
AND DOING THESE TYPES OF
ANALYSIS AND NOW LOOKING AT THIS
IN THE CONTEXT OF ADULTS.
WHEN YOU HAVE A TOOTH EXTRACTED,
SAVE THAT TOOTH.
WE CAN USE THAT TO BUILD YOUR
EXPOSURE PROFILE.
THERE'S ALSO COOL WORK GOING ON
WITH MEASURING THE EXPOSOME AND
ADIPOCYTES WHICH ARE STABLE
TISSUE.
THE NOW WITH THE EMERGING
TECHNOLOGIES THEY ARE ALLOW US
TO DO UNBIASSED ANALYSIS OF C
CHEMICALS AND LOOK AT SOCIO
ECONOMIC LEVELS IMPACTING THE
EXPOSOMICS AND WE'RE LOOKING RAT
EPIGENETIC MARKS OF ADVERSE
EVENTS IN SOMEBODY'S LIFE,
LIVING IN A POOR NEIGHBORHOOD,
HAVING MATERNAL STRESS.
THERE'S A RICHER DATABASE OF
MARKS THAT ALLOWS US TO
INTEGRATE THOSE TYPES OF
EXPOSURES AND DOING THIS WITH
CHEMICALS AS WELL.
EPIGENETICS IS A COOL POTENTIAL
TO INTEGRATE EXPOSURES NOT ONLY
WITHIN A SPECIFIC WINDOW OF TIME
BUT MAYBE ACROSS THE LIFE TIME
AS WELL.
>> FIRST'S AMAZING TO SEE THIS
TAKING SLAP.
-- SHAPE.
IT'S TIME.
THE BRAIN AND NERVOUS SYSTEM ARE
PROBABLY MORE AFFECTED BY THE
ENVIRONMENT.
I AGREE THERE'S CLINICAL DATA
COMING OUT.
IT'S FANTASTIC WE HAVE CRITICAL
ASSOCIATIONS YOU'RE SPEAKING TO
NOW.
I WAS THINKING ABOUT THE
MECHANISTIC WORK BEING DONE
ACROSS THE NINDS FUNDED LABT
RIPS INTRAMURALLY THAT CONTAIN A
TREASURE TROVE OF AMAZING ANIMAL
MODELS AND PRE-CLINICAL MODELS
AND WORK AIMED AT THE TARGET OR
THE DISEASE PROCESS THAT YOU
HYPOTHESIZED BASED ON CLINICAL
ASSOCIATIONS IS THE AFFECT OF
PESTICIDES IN KIN SONS AND
MITOCHONDRIAL TALKS AND LOOKING
AT THE TARGETS OF BIOMARKERS OR
MECHANISMS.
THERE COULD BE ALREADY EXISTING
EXPERTISE.
IT'S ACROSS EVERY DISEASE
PROCESS.
ASKING THE LABORATORY TO WORK
WITH THE TARGET OR HAVE THE
MODEL TO STUDY IT FOR THAT
CAUSALITY CORRELATION.
>> EXCELLENT POINT.
THAT IS ENCAPSULATED IN THE FULL
REPORT.
THANK YOU FOR BRINGING THAT UP
AND EMPHASIZING THIS AND WE ARE
AT A GREAT TIME TO DO THIS.
THERE'S SO MANY OF THESE MODELS
DEVELOPED NOW TO LOOK AT CAUSAL
MECHANISMS LINKING TO EFFECTS
AND OUTCOME.
PERFECT SUGGESTION.
THANK YOU.
>> THIS ONLY THING I CAN SAY IS
I'LL SKIP THE OTHER STUFF THINGS
LIKE ECHO AND AECD AND A LOT OF
THESE ARE DOING THIS ALREADY.
THINGS ARE HAPPENING NOW.
IF THINGS ARE HAPPENING, EUROPE,
JAPAN, A LOT IS GOING ON THERE.
I'VE BEEN THERE.
AND EARLIER WHEN WE WERE TALKING
ABOUT PREVENTION AND HOW THAT'S
GOING TO BE IMPORTANT SEEMS LIKE
THREES SOMETHING WE CAN DO TO
HELP GET THIS EXPOSOME RESEARCH
OUT.
>> I AGREE.
THIS IS REALLY THE TRUE PROMISE
OF EXPOSOMIC RESEARCH IS
PREVENTION.
IT'S BETTER TO PREVENT DISEASE
THAN TO TREAT IT.
THERE'S A POTENTIAL TO HAVE A
SIGNIFICANT IMPACT ON THE BROAD
SWATH OF POPULATION THAT DOESN'T
REQUIRE AN INDIVIDUAL TO CHANGE
THEIR LIFESTYLE OR MAYBE IT DOES
BUT THERE'S POTENTIAL TO REDUCE
HEALTH CARE COSTS BY LOOKING AT
THE NEURAL EXPOSOME.
>> NOW, AS A FORMAL WORKING
GROUP OF COUNCIL WE'LL ASK FOR A
MOTION TO MOVE FORWARD THE
RECOMMENDATIONS.
IS THERE A MOTION?
SO MOVED.
>> IS THERE A SECOND?
>> SECOND.
>> OKAY.
GREAT.
WE'LL DO THE CALL OUT VOTING AS
WE DID IN THE '50s.
AMY, VOTING AMY.
>> APPROVE.
>> BOB.
>> APPROVE.
>> JEN.
>> APPROVE.
>> JANE.
>> APPROVE.
>> JIM MU.
>> APPROVE.
>> JOHN.
>> APPROVE.
>> LOUISE.
>> APPROVE.
>> GINA.
>> APPROVE.
>> GEM ENNI.
>> APPROVE.
>> JIM.
>> SO MOVED.
>> APPROVED AND ACCEPTED.
WE HAVE A REPORT OUT NOT AN FY
BUT UPDATE.
RICHARD IS OUR DIRECTOR OF
OFFICE AND GLOBAL HEALTH
DISPARITIES.
>> GOOD AFTERNOON, EVERYONE.
THANK YOU.
THIS IS NOT A REPORT OUT THIS
TIME.
IT'S MY HONOR TO PRESENT WE LEAD
THE COORDINATION AND REPORTING
AND INITIATIVES RELATED TO
NATIONAL AND INTERNATIONAL
RESEARCH ON DISPARITIES AND
INEQUITIES IN NEUROLOGICAL
DISEASES.
DR. SANKAR WILL BE PRESENTING
TOMORROW AND SHE'LL TALK MORE
ABOUT THAT TOMORROW.
NINDS HAS BEEN DOING GLOBAL
HEALTH RESEARCH OVER 20 YEARS.
STACY HAS BEEN HERE MOST THE
TIME.
SHE HAS HISTORICAL KNOWLEDGE
RELATED TO GLOBAL HEALTH AND
LEADING A PLANNING PROCESS WE'RE
EMBARKING ON AND THAT'S WHAT
I'LL TALK ABOUT TODAY AND GO
INTO MORE.
WHEN WE TALK ABOUT GLOBAL HEALTH
SO THAT'S SORT OF OUR PRIMARY
GOAL IN BUILDING CAPACITY
THROUGH INVESTMENT IN RESEARCH
AND TRAINING TO ADDRESS PUBLIC
HEALTH CHALLENGES RELATED TO
NEURO LOGICAL DISORDERS.
WHEN WE THINK OF GLOBAL HEALTH
AND HAVE SPOKEN WITH OTHER
LICE
LICENSEES IN TERMS OF GLOBAL
HEALTH RESEARCH WE SEPARATE THAT
IS SEPARATED FROM INTERNATIONAL
RESEARCH WITH HIGH INCOME
COUNTRIES.
RAND THE REASON FOR RESEARCH IN
LOW AND MIDDLE INCOME COUNTRIES,
NEURO LOGICAL CONDITIONS IT'S
THE NUMBER ONE CAUSE OF DISEASE
BURDEN WORLDWIDE.
IN 2021 ABOUT 3.4 BILLION
INDIVIDUALS HAD CONDITIONS
AFFECTING THE NERVOUS SYSTEM.
THINK ABOUT THAT.
THAT'S 43% OF THE WORLD'S
POPULATION.
I HAVE LISTED ON THE RIGHT SIDE
OF THE SLIDE THE PREDOMINANT
DISEASES THAT IMPACT PEOPLE.
STROKE IS THE MOST COMMON CAUSE
OF DEATH WORLDWIDE.
WE HAVE OTHER DISEASES LISTED ON
THE SLIDE.
IN TERMS OF THE DISABILITY
ADJUSTED LIFE YEARS THAT'S 443
MILLION DAILY ASSOCIATED WITH
NEUROLOGICAL DISEASES.
THAT'S HUGE.
THE BURDEN IS MASSIVE.
THAT MAKES AND THOSE OCCUR IN
LOW AND MIDDLE INCOME COUNTRIES.
AND IF YOU LOOK AT THE
PARTICULAR GRAPH YOU CAN SEE THE
LARGE CIRCLES.
THOSE ARE LOCATED PRIMARILY IN
LOW AND MIDDLE INCOME COUNTRIES
HAVE THOSE AREAS.
SO AS DR. KOROSHETZ MENTIONED
EARLIER AND I WANT TO PREFACE
THAT DR. KOROSHETZ HAS BEEN VERY
SUPPORTIVE OF THE WORK WE'RE
DOING IN OUR OFFICE BOTH IN
HEALTH EQUITY AS WELL AS GLOBAL
HEALTH AS WELL AS DR. CLINTON
WRIGHT.
SUPPORTIVE OF THE WORK WE'RE
DOING WE WOULD NOT BE ABLE TO DO
IT WITHOUT THE SUPPORT OF OUR
LEADERSHIP.
SOME OF THE AREAS WE LIKE TO
EMPHASIZE AND WE'RE PUTTING
TOGETHER A GROUP OF THE ADVISORY
COUNCIL AND WOULD LIKE
VOLUNTEERS TO WORK WITH US ON
THIS.
WE HAVE A SENIOR ADVISER ON THE
COUNCIL WORKING WITH US ON OUR
PLANNING PROCESS.
HE WILL BE ON THIS AND WE'D LIKE
TO HAVE TWO CO-CHAIRS.
WE HAVE A CO-CHAIR FROM JOHNS
HOPKINS AND WOULD LIKE 12
MEMBERS FOR OUR NATIONAL
ADVISORY COUNCIL AND REPRESENT
THE GLOBAL ENVIRONMENT AS WELL
AS MULTIPLE NEUROLOGICAL DISEASE
AREAS.
E-MAIL BACK TO DR. KOROSHETZ IF
YOU'RE INTERESTED IN WORKING IN
THE GROUP WE THINK WILL BE
IMPACTFUL.
PUTTING THOSE TWO TOGETHER WORK
I'VE DONE OVER THE YEARS I HAVE
NOT THOUGHT THEIR MASSIVE
CONNECTION AND THERE'S SOCIAL
DETERMINATES OF HEALTH THAT
IMPACT THE U.S. AS WELL AS
GLOBALLY.
IN ADDITION TO MULTIPLE OTHER
FACTO
FACTORS WE LIKE TO BUILD
CAPACITY AT VARIOUS LEVELS AND
BUILDING BILATERAL PROGRAMS WE
HAD OVER 30 RESPONSES WHICH IS A
LOT FOR AN RFI AND INDIVIDUALS
AND ORGANIZATIONS AND
INSTITUTIONS AND DOMESTIC
RESPONSES AS WELL.
AND WE'RE ALSO IN THE PROCESS OF
DOING PORTFOLIO ANALYSIS OF
EVERYTHING THE INSTITUTE FUNDED
IN THE LAST FIVE TO 10 YEARS
THAT'S ONGOING.
WE'LL PUT TOGETHER THE WORKING
GROUP AND START AN NINDS WIDE
AND TRANS-NIH WORKING GROUP AS
WELL AND MEET WITH OTHER I.C.s
SO THAT THE PROCESS THEY'RE
GOING THROUGH CAN INFORM OR
THEY'VE GONE THROUGH CAN INFORM
WHAT WE ARE DOING.
WE MET WITH DR. KOROSHETZ WITH
THE NEW HEAD OF THE FOGARTY
INTERNATIONAL CENTER AND THEY'RE
GOING THROUGH THE STRATEGIC
PLANNING PROCESS FOR HEALTH
EQUITY.
WE HAVE A WEEKLY NEWSLETTER WE
SEND OUT WITH INFORMATION ABOUT
GLOBAL HEALTH AND HEALTH
INEQUITIES.
WE HAVE A SOCIAL SITE SO IF YOU
WANT TO FOLLOW US, PLEASE DO
THAT AND LET DR. KOROSHETZ KNOW
IF YOU'RE INTERESTED IN
PARTICIPATING IN THE WORKING
GROUP OF THE NATIONAL ADVISORY
COUNCIL AND IF THERE ARE ANY
QUESTIONS, I'M HAPPY TO ADDRESS
THOSE IF WE HAVE TIME.
>> THANK YOU.
IN THE LIST OF THE CONDITIONS
YOU NOTED I WOULD ASSUME HEAD
TRAUMA WOULD BE ON THIS AND
SOMETHING THE W.H.O. NOTED AS
UNDER ADDRESSED ISSUES OR MAYBE
I'M MISSING WHERE IT'S BEING
REPRESENTED.
>> GREAT POINT.
I MET WITH THE WORLD HEALTH
ORGANIZATION AND MOST PEOPLE
THEY ARE MET WITH WERE IN THE
AREA OF BRAIN INJURY.
IT IS A MAJOR GLOBAL PROBLEM.
WE HAVE A LARGE PORTFOLIO TO
FUND THE TRAUMATIC BRAIN INJURY.
IT'S A MAJOR PROBLEM AND ONE OR
TWO DISEASES AFTER THIS.
>> TO CLARIFY, TO BE SURE I JUST
SAW SOMETHING, IS THIS GROUP THE
GLOBAL BURDEN OF DISEASE GROUP
THAT REPORTS ON THESE KINDS OF
INCIDENT PREVALENCE AND
BASICALLY THEY REPORT OUT TRAUMA
BUT DON'T NOTE HEAD INJURY FROM
OTHER KIND OF TRAUMA.
IT'S REALLY BIG AND THE
SUSPICION IS THERE'S NO WAY OF
KNOWING FOR SURE.
>> THE WORKING GROUP KEPT THE
TBI COMPONENT IN MIND.
WE WERE DISCUSSING MAYBE IT'S
THE CLASSIFICATION OF WHAT'S
BEING ATTRIBUTED.
>> OFTEN STROKE IS CONSIDERED A
BRAIN INJURY FOR SOME PEOPLE.
SPECIFICALLY WITH TRAUMATIC
BRAIN INJURY IT'S A VERY BIG
DISEASE PROCESS WE SEE IN THE
GLOBAL SPACE.
SO WE DO FUND SEVERAL PROJECTS
IN THAT AREA.
THAT'S A GREAT QUESTION.
>> AS WOE THINK OF THE
INITIATIVES WE'RE TALKING ABOUT
TODAY AROUND NEW TECHNOLOGY,
GENETIC THERAPIES, I'M AFRAID
WE'RE ALMOST INCREASING INEQUITY
ACROSS THE WORLD AS THEY'RE MORE
ACCESSIBLE TO FIRST-WORLD
COUNTRIES AROUND THE WORLD.
THERE'S THE DANGER OF THE MORE
BOUTIQUE MEDICINE.
ARE THERE WAYS TO MORE
PROACTIVELY ADDRESS THESE AND
THINK ABOUT WAYS OF CALLING THIS
OUT AND THINK OF BEST PRACTICES
AS ONE TRIES TO ADDRESS
DISPARITY?
>> WE'VE ADDRESSED DISPARITIES
CREATED WITH NEW TECHNOLOGY AND
ALSO WOULD LIKE TO MAKE THE
POINT THAT WE COULD ACTUALLY
LEARN IN THE U.S. ABOUT
PROVIDING MEDICAL CARE TO
CERTAIN POPULATIONS
DISADVANTAGED TO HEALTH CARE AND
SOME NEWER TECHNOLOGIES ARE NOT
ACCESSIBLE OR AFFORDABLE TO
PEOPLE WITHIN THE UNITED STATES.
WE ARE LOOKING INTO LOW AND
MIDDLE INCOME COUNTRIES AND LOOK
BEING AT HOW MEDICINE FOR STROKE
AND OTHER DISEASES NEUROLOGICAL
DISEASES COULD BE IMPLEMENTED IN
THE UNITED STATES AS WELL.
WE DO HAVE -- WE HAVE THIS
DICHOTOMY IN THAT WE HAVE
EXPENSIVE TECHNOLOGIES COMING
OUT BUT HAVE PEOPLE WHO DON'T
HAVE ACCESS.
WE'RE THINKING OF BOTH ASPECTS
OF THAT POINT.
>> I'LL REFER BACK TO THE PAPER
FYODOR MENTION ED AND INCLUDE
DEVELOPING GMP FACILITIES IN
UNDER DEVELOPED COUNTRIES TO
MANUFACTURE GENOMIC THERAPIES
AND DELIVER AT COST.
THERE'S SHAREHOLDERS AND THERE'S
VALUE-BASED PRICING TO GET AS
MUCH AS MONEY AS YOU CAN GIVEN
THE SEVERITY OF DISEASE.
THAT'S NOT SCALABLE ACROSS THE
WORLD.
IF THERE WAS A DIFFERENT SYSTEM
WHERE THESE THERAPIES COULD BE
DEVELOPED THROUGH ACADEMIC
PARTNERSHIPS AND NONPROFITS THEY
WOULD NOT BE CLOSE TO WHAT THEY
COST.
>> IS THERE EMPHASIS ON THE
HUMAN MIGRATION FACTOR AND
REFUGEE CRISES AROUND THE WORLD
THAT ARE PARTICULARLY VULNERABLE
POPULATIONS?
>> THAT'S ANOTHER GREAT POINT.
AND THERE'S A SENIOR ADVISER
FROM VANDERBILT AND SAID ONE
REASON THE U.S. SHOULD BE
INVESTMENTS IN THIS IS BECAUSE
THE LARGE BURDEN OF NEUROLOGICAL
DISEASE IN THE LOW AND MIDDLE
INCOME COUNTRIES WHERE THE
AVERAGE AGE IS MILLIONS OF
PEOPLE IN THEIR 20s UNLESS WE
DEAL WITH THESE AND WE CAN HAVE
ISSUES IN THE FUTURE WITH PEOPLE
WITH LARGE NEUROLOGICAL DISEASES
FOR LARGE PERIOD OF TIME
MIGRATING TO THE U.S. AND OTHER
PLACES AND THE IMPACT OF THAT AS
WELL.
AND THIS COULD HAVE BEEN
PREVENTIBLE.
FOCUSSING ON WHY THIS IS SO
IMPORTANT SPECIFICALLY IN TERMS
OF NEUROLOGICAL DISEASE AND THE
BURDEN THAT GOES WITH THIS.
>> THERE'S AN OFFICE OF HEALTH
DIPLOMACY WHICH THINK OF THESE
THINGS IN THE 30,000 FOOT VIEW.
>> THANK YOU.
WE LOOK FORWARD TO VOLUNTEERS
AGAIN.
>> THANK YOU FOR THE UPDATE.
I BELIEVE WE'LL BE MOVING TO OUR
FINAL ITEM OF THE OPEN SESSION
WHICH IS CONCEPT CLEARANCE.
NOW, WHAT WE DO IS WE ALWAYS
CLEAR OUR CONCEPTS BOTH NEW AND
RE-ISSUES TO THE COUNCIL.
MANY PRESENTATIONS ARE IN THE
WRITTEN FORM AND IN THE COUNCIL
BOOK HOWEVER, FROM TIME TO TIME
WE FIND SOME WORTHY THAT WE
THINK ARE A NEW PROGRAM OR
PEOPLE WILL BE INTERESTED IN
HEARING ABOUT AND THIS TIME WE
HAVE TWO WE'LL HAVE STAFF
PRESENT AND WE'LL PLAY IT BY EAR
WE CAN DO A SINGLE VOTE AT THE
END BUT THE FIRST PROGRAM WE'LL
HEAR ABOUT IS FROM FROM THE
DIVISION OF NEUROSCIENCE.
GLEN.
>> GOOD AFTERNOON.
I'M THE PROGRAM DIRECTOR AT THE
GENETICS CLUSTER AND COVER
INHERITED AND NEUOPATHIES AND
HAVE A NEW INITIATIVE ON
GUILLAIN-BARRE SYNDROME AND
PARALYSIS AND OTHER SYMPTOMS.
GBS IS THE ACUTE ONSET, RAPIDLY
PROGRESSING FORM IN SOME CASES
TRIGGERED BY GASTROENTERITIS OR
RESPIRATORY INFECTION AND LEAD
TO ANTIBODIES THAT CROSS REACT
WITH INFLAMMATION.
A SIGNIFICANT PORTION OF DBS
CASES MAY EXPERIENCE FULL
RECOVERY WITHIN A YEAR OR SO BUT
THERE'S ALSO A SMALLER PORTION
THAT COULD LEAD TO RESPIRATORY
FAILURE OR HAVE LIFE LONG
DISABILITY.
SO CIPD IS A MORE CHRONIC
CONDITION OF PERIPHERAL NERVE
INFLAMMATION AND TRIGGERS
ASSOCIATED AND THE TREATMENTS
FOR THE CONDITIONS ARE
RELATIVELY NON-SPECIFIC.
THINGS LIKE IBIG, PLASMA PARESIS
AND INEFFECTIVE FOR A NUMBER OF
INDIVIDUALS AFFECTED.
WE LOOKED BACK AT APPLICATIONS
RECEIVED OVER THE PAST FOUR
YEARS THAT MENTIONED GBS AND
CIDP.
THERE'S FEW APPLICATIONS WE'RE
RECEIVING ON THESE CONDITIONS
PART MAY BE IT'S A SMALL COHORT
OF INVESTIGATORS THAT SEND
APPLICATIONS ON THE TOPICS.
WE'RE ENVISION IING AN INITIATI
THAT FIRST FOCUSES ON ACCELERATE
BEING PROGRESS TOWARDS
UNDERSTANDING AND TREATING THESE
CONDITIONS.
AND WE'D ALSO LIKE TO FIND WAYS
TO ATTRACT INVESTIGATORS WITH
APPROPRIATE EXPERTISE FROM
NEIGHBORING FIELDS TO ENCOURAGE
THEM TO FOCUS ON THESE AND
FOCUSSING ON EXPLORATORY AND
DEVELOPMENT STUDIES, ANIMAL
MODELS, SPECIMENS OR DATA FROM
PATIENTS BUT WE'RE NOT PLANNING
ON SUPPORTING ANY LARGE COHORT
STUDIES OR CLINICAL TRIALS.
HERE'S EXAMPLES OF TOPICS THAT
MIGHT FIT IN THE INITIATIVE.
BETTER UNDERSTANDING THE
CELLULAR AND HUMORAL MECHANISMS
LEADING TO DEMILE --
DEMYELINATION OR EVALUATE THE
EFFECTIVENESS OF NEW TREATMENTS
AND THERE'S A NEED FOR
BIOMARKERS SUCH AS PROGNOSTIC
BIOMARKERS THAT CAN BE USED RAND
AND THIS IS A BRIEF OVERVIEW OF
WHAT WE'RE ENVISIONING.
>> COULD THERE BE A CLINICAL
TRIAL THAT IS BASED ON RATIONALE
OF SMALL SCALE?
>> NINDS HAS FUNDING
OPPORTUNITIES THAT WOULD INVITE
SMALL SCALE CLINICAL TRIALS.
WE HAVE EXPLORATORY CLINICAL
TRIALS AND OTHER FUNDING
MECHANISMS FOR LARGER CLINICAL
TRIALS.
WE ALSO HAVE AN INITIATIVE THAT
WILL COME UP IN THE RENEWAL
CLINICAL TRIAL READINESS PROGRAM
FOR RARE DISEASES THAT SUPPORT
COHORT STUDIES AIMED AT
BIOMARKER AND OUTCOME MEASURE IN
CLINICAL VALIDATION.
WHAT WE'RE THINKING HERE IS MORE
EXPLORATORY STUDIES AND LOOK AT
OTHER RESEARCH.
>> AND TO ADD WE HAD A UNIQUE
OPPORTUNITY TO DO THIS WORK SO A
FEW WORDS.
>> SO NIH SURPRISINGLY AND NINDS
SPECIFIC
SPECIFICALLY WITHOUT ADVERTISING
GETS DONATIONS FROM GRATEFUL
PATIENTS AND FAMILIES AND THIS
INITIATIVE IS FUNDED BY A
DONATION TO THE FUND THAT'S
CONDITIONAL IT BE USED FOR GAMMA
RAY.
-- GUILLAIN-BARRE.
WE DON'T GET A LOT OF
INFORMATION ABOUT THE DONOR BUT
IT'S APPRECIATED AND EVERY DAY
OR TWO WE GET A CHECK IN THE
MAIL IN SOMEBODY.
USUALLY FROM THE DEATH OF
SOMEONE WITH A NEUROLOGICAL
DISEASE.
WE TRY TO USE THAT MONEY REALLY
WELL.
THANKS, GLEN.
>> SO, NEXT WE'LL HEAR FROM OUR
OFFICE OF PROGRAM TO ENHANCE HE
NEUROSCIENCE WORKFORCE.
NICE IT SEE YOU.
>> IT'S NICE TO BE SEEN.
THANK YOU FOR YOUR ATTENTION.
I'M MARGUERITE MATHEWS AND I'M
HERE TO TALK ABOUT A RE-ISSUE
FOR A LIMITED COMPETITION
PROGRAM TO PROVIDE
POSTBACCALAUREATE EXPERIENCES.
AND THREE YEARS AGO I WAS BEFORE
YOU SO TIME PASSES QUICKLY.
THE PROGRAM WAS FIRST CONCEIVED
BY OUR OFFICE AND TRAINING
WORKFORCE AT THE TIME LED BY
DR. STEVE CORN TO THINK ABOUT
HOW TO FILL A GAP THAT WAS
MISSING IN THE NEUROSCIENCE
WORKFORCE.
WE WERE SEEING OR HEARING A LOT
OF FEEDBACK THROUGH OUR
STRATEGIC PLANNING THROUGH SOME
OF OUR OTHER PROGRAMS ABOUT NOT
BEING ENOUGH OPPORTUNITIES FOR
POSTBACCALAUREATE EXPERIENCES
AND MANY WERE NOT ABLE TO GET
INTO GRADUATE SCHOOL BECAUSE WE
DIDN'T HAVE THE OPPORTUNITIES
AND WANTED A PROGRAM THAT
DOESN'T FURTHER DELAY THE
OPPORTUNITY FOR FOLKS TO GO IN
THE Ph.D. PROGRAMS.
WE DECIDED TO ISSUE A LIMITED
COMPETITION, R25 WITH ACTIVE T32
PROGRAMS TO PROVIDE NEUROSCIENCE
TRAINING EXPERIENCES BECAUSE
THESE T32 PROGRAMS ARE ALREADY
VETTED AND HAVE BEEN PEER
REVIEWED AND DOING EXCELLENT
WORK IN THE TRAINING SPACE.
WITH THE T32 PROGRAMS HAVE THE
INFRASTRUCTURE AND HAVE THE
PLANNING TO BE ABLE TO PROVIDE A
MEANINGFUL TWO-YEAR RESEARCH
EXPERIENCE FOR THE
POSTBACCALAUREATE TRAINEES AND
WANTING TO MAKE SURE WE'RE
TARGETING TRAINEES FROM
INSTITUTIONS WHERE THEY'RE NOT
ALREADY GETTING SUBSTANTIAL
RESEARCH EXPERIENCES.
THINK LIBERAL ARTS COLLEGES, AND
THIS IS INTENDED TO HAVE A
TWO-YEAR EXPERIENCE AND THEN
BECOME COMPETITIVE TO ENTER INTO
Ph.D. PROGRAMS.
THE PROGRAM COSTS RELATIVELY THE
SAME.
THE IDEA IS TO PROVIDE MOST OF
THIS GOING TOWARDS THE SALARY
FOR TRAINEES COVERING THEM FOR
TWO TOTAL YEARS.
SO YEARS 1 AND 2 WOULD BE AROUND
$225,000 IN DIRECT COSTS AND
THEN IN THE MIDDLE YEARS, TWO
AND THREE THERE WOULD BE ALMOST
A DOUBLED COST BECAUSE IT'S A
TWO-YEAR PROGRAM YOU'RE ALSO
HAVING OTHER COHORTS COMING IN.
SO THERE WOULD BE IN THOSE
MIDDLE YEARS TWO COHORTS AT TO
THE SAME TIME BEING FUNDED.
CURRENTLY SINCE THE FIRST ROUND
CAME IN FY2022, THE YEAR ROUND
ACTIVE PROGRAMS STARTED DECEMBER
2022.
AND THEY'RE ALL CURRENTLY NOW IN
YEAR TWO OF FUNDING.
THEY'VE HAD ONE COHORT
SUCCESSFULLY FINISH ONE YEAR AND
JUST BROUGHT ON THE SECOND
COHORT THIS SUMMER AND THEY'LL
BE FINISHISHING -- FINISHING UP
OF THE AWARD IN THE FALL/WINTER
OF 2026.
WE'RE DOING A GREAT JOB OF
FUNNELING FOLKS IN THE
NEUROSCIENCE PATHWAY AT HIGH
SCHOOL AGE, UNDERGRADUATE.
WE HAVE A LOT OF OPPORTUNITIES
AT THE DOCTORAL, NEW FACULTY
LEVEL.
THERE'S SOMETHING LACKING IN THE
POSTL
POSTLACK -- POSTBACCALAUREATE
SPACE AND THERE'S THE PREP
PROGRAM.
ONE HAS.
THE ONLY OTHER EXISTING PROGRAM
FOR THE NEUROSCIENCES.
I WOULD LIKE TO TELL YOU IT'S
GOING PRETTY WELL.
WE HAVE SIX FUNDED PROGRAMS AT
HARVARD, UNIVERSITY OF IOWA AND
KENTUCKY AND WASHINGTON
UNIVERSITY IN ST. LOUIS.
EACH SITE WILL BE EXPECTED TO
RECRUIT SCHOLARS FOR COHORT.
THEY HAVE FUNDING FOR THREE
COHORTS TOTAL.
FOUR OF THE SIX PROGRAMS MET THE
GOAL FOR COHORT 1 AND MET THE
GOAL FOR COHORT 2.
I HAVEN'T HEARD FROM THE OTHER
TWO PROGRAMS JUST YET SINCE THEY
HAVEN'T PUT IN THEIR RP PRs.
IN GENERAL THEY ARE DOING THEIR
BEST TO MAKE SURE HAVE FOLKS
THAT MEET THE CRITERIA WE'RE
ASKING FOR AND THEY'RE ALSO
AFTER THE FIRST COHORT ALL THE
PROGRAMS STARTED TALKING TO EACH
OTHER AND SHARE INFORMATION
ABOUT STUDENTS APPLYING SAYING
WE DON'T HAVE THE SPACE FOR THIS
TRAINEE BUT THERE'S STILL
INTERESTED IN THE PROGRAM, WOULD
YOU BE ABLE TO ACCEPT THEM AND
IT SEEMS TO BE WORKING WELL.
I APOLOGIZE THESE ARE ESTIMATES.
IT'S JUST THE NATURE OF THE
FUNDING CYCLE.
SO THIS SLIDE IS JUST TO SHOW
YOU THESE PROGRAMS ARE
RECRUITING FROM INSTITUTIONS
THAT DO NOT HAVE THIS RESEARCH
CAPACITY.
SO LARGELY INSTITUTIONS THAT
DON'T HAVE ANY SORT OF GRADUATE
LEVEL HAPPENING AND MANY ARE
LIBERAL ARTS AND SOME ARE IN
RURAL AREAS.
AND NOT A LOT OF DATA JUST YET
BUT HAPPY TO SEE THEY'RE GOING
FAR AND WIDE TO MAKE SURE THE
OPPORTUNITY IS AVAILABLE TO
THOSE WHO NEED IT.
THERE ARE THREE SLIDES WORTH OF
INFORMATION PROVIDED BY OUR
PROGRAM DIRECTORS TO SHOW YOU
HOW WELL THINGS SEEM TO BE
WORKING.
UNFORTUNATELY BECAUSE OF THE
TIMING, THE FIRST ISSUE WAS ONLY
FOUR YEARS AND SO THERE'S NOT
ENOUGH TIME TO DECIDE HOW WAS IT
WORKING, THE FIRST COHORT HASN'T
FINISHED THEIR SECOND YEAR YET.
THEY'LL ONLY START APPLYING TO
GRADUATE SCHOOL THIS FALL.
SO WE WON'T KNOW HOW MANY WILL
HAVE GONE TO THE PROGRAM BUT
IT'S WORTH INVESTING IN.
THEY'RE WORKING WELL TOGETHER
WITH THEIR T32 PROGRAMS.
OTHER R25 AND SIMILAR PROGRAMS
AID AT THE DOCTORAL LEVEL AND
MANY INSTITUTIONS ARE STARTING
TO PROVIDE IN-KIND
ADMINISTRATIVE TO SUPPORT TO
MAKE SURE THEY'RE SUCCESSFUL AND
P.I.s ARE IN TOUCH MAKING SURE
THEY'RE GOOD STEWARTS OF THE
FUNDING OPPORTUNITIES AND BEING
RESPONSIVE TO THE NEEDS OF
TRAINING -- TRAINEES WHO DON'T
HAVE EXPERIENCE AND MANY HAVE A
CULTURE SHOCK GOING IN THESE
ENVIRONMENTS.
I'M PLEASED TO SEE HOW IT'S
WORKING OUT.
AND WE CAN IMPROVE HOW WE'RE
APPROACHING THIS AND THE GENERAL
IDEA IS TO REQUEST FUNDING AT
THE SAME LEVEL WE'VE HAD IN THE
PAST TO GIVE PROGRAMS ANOTHER
OPPORTUNITY TO SHOW HOW WELL
THEY'RE DOING AND HOW THEY'LL
CONTINUE TO MOVE THESE SCHOLARS
TO THE NEXT PHASE OF THE
PATHWAY.
I ENCOURAGE YOU TO READ SOME
HIGHLIGHTS.
>> QUICK QUESTION.
WAS IT THE SAME SITE THAT HAD
TROUBLE RECRUITING?
THE SAME TWO?
>> NO, I KNOW THEY'RE NOT THE
SA
SAME.
ONE HAD TO TAKE A LEAVE OF
ABSENCE AND WEREN'T ABLE TO
INTERN THOUGH THEY HAD BEEN
ACCEPTED.
ANOTHER PROGRAM WE WERE ONLY
ABLE TO GET THREE TO COMMIT.
BOTH OF THOSE PROGRAMS APPROVED
FIVE STUDENTS IN THE SECOND
YEAR.
I'M ASKING THEM ON A SHORT TIME
LINE CAN YOU GIVE ME YOUR
INFORMING?
>> THANKS FOR --
GIVE ME YOUR INFORMATION?
>> ARE THE T32s SOMETHING YOU'LL
WANT TO CONTINUE?
>> THE REASON IT'S A LIMITED
COMPETITION IS BECAUSE IT'S A
PILOT PROGRAM.
IN SOME WAYS IT'S RISKY.
BECAUSE THESE TRAINEES HAVE NOT
HAD PREVIOUS RESEARCH
EXPERIENCE, WE'RE INVESTING
HOPING THEY'RE GOING TO MAKE THE
TRANSITION TO GRADUATE SCHOOL
BUT WHAT IF THEY FIND IT'S NOT
THE RIGHT OPPORTUNITY FOR THEM?
IS THERE A WAY TO CREATE A
PROGRAM WHAT IS GOING TO
INCREASE THE LIKELIHOOD OF THE
TRAINEES GETTING THE EXPERIENCE
THEY NEED TO CONVERT TO GRADUATE
SCHOOLS?
WE LIMITED IT AND TO MAKE SURE
THESE ARE REALLY WELL RECOGNIZED
T32 TRAINING PROGRAMS THAT HAVE
EVERYTHING NEEDED TO MAKE SURE
THESE TRAINEES HAVE NOT JUST
RESEARCH EXPERIENCE BUT
PROFESSIONAL DEVELOPMENT, CAREER
DEVELOPMENT OPPORTUNITIES WHERE
THEY'RE GOING TO PARTICIPATE IN
POSTER SESSIONS AND ABLE TO
INTERACT WITH OTHER FACULTY,
KNOW WHAT IT'S LIKE TO BE IN
THAT ENVIRONMENT TO MAKE GOOD
DECISIONS WHETHER OR NOT THEY
WANT TO ENTER INTO THAT NEXT
PART OF THAT PATHWAY.
>> DO YOU CONSIDER IT A SUCCESS
OR FAILURE IF THE STUDENTS WIPED
UP GOING TO MEDICAL SCHOOL?
>> WE HAD THE CONVERSATION WITH
PROGRAM DIRECTORS AND THEY'RE
REALLY KEEN ON SCREENING FOR
WHAT THEIR TRUE INTERESTS ARE.
WE CAN'T HELP IF A STUDENT
DECIDES LATER ON IF THAT'S WHAT
THEY ARE WANT TO DO.
THEY'RE NOT SUPPOSED TO BE
INCLUDING TRAINEES WHO HAVE AN
INTEREST IN MEDICAL SCHOOL LIKE
RIGHT OFF THE BAT.
MANY START TO LOOK AT INTERESTS.
I'VE HAD ONE-ON-ONE, THE TRAINEE
CAME IN.
THEY SAID THEY WERE INTERESTED
IN RESEARCH AND TWO MONTHS IN
THEY SAID I WANT TO GO TO
MEDICAL SCHOOL AND HELP THE
STUDENT TRANSITION OUT TO FIND
ANOTHER LAB WHERE THEY ARE CAN
STILL GAIN RESEARCH EXPERIENCE
AND HAVE OTHER SUPPORT TO BE
ABLE TO MOVE ON TO PURSUE THEIR
THE MEDICAL FIELD.
>>
>> WE HAVE ONE OF THESE PROGRAMS
AT WASH U.
I THINK THE CONCERN THAT WAS
STATED WHAT IF THEY DECIDE THIS
IS NOT FOR THEM, THAT'S A KNOCK
AGAINST SOMEONE COMING FROM ANY
UNDER RESOURCED INSTITUTION.
I THINK THAT'S NOT A PROBLEM.
AND TO SAY IF SOMEONE DECIDES TO
GO TO MEDICAL SCHOOL IS A
PROBLEM WOULD BE SILLY
GENERALLY.
BUT AND I'M NOT A PHYSICIAN OR
ADVOCATING BUT THIS LAPPENS.
BEING THE TWO YEAR PROGRAM IS
SUPER IMPORTANT.
THE STUDENTS COME IN AND THEY'RE
A COUPLE MONTHS IN AND NOW
APPLYING TO GRADUATE SCHOOL
WON'T WORK.
SEEING THE PROGRESS OF THE
STUDENTS FROM YEAR ONE TO TWO
HAS BEEN IMPRESSIVE.
THE PROGRAM AS STRUCTURED IS
SUPER IMPORTANT BECAUSE IT'S
LIKE BEING IN GRADUATE SCHOOL.
THEY WILL KNOW WHAT THEY'RE
GETTING INTO.
>> THE IDEA IS THEY'RE MAKING
SURE THE STUDENTS UNDERSTAND
WHAT THEY'RE GETTING.
YOU'RE GETTING EXPERIENCE LIKE A
Ph.D. PROGRAM WHICH IS WHAT IS
DIFFERENT THAN TO GET INTO AN
M.D. PROGRAM AND SOME IS TE
TEMPERING EXPECTATIONS AND THIS
IS WHAT OUR RESOURCES ARE GOING
TOWARDS.
IT SEEMS TO BE WORKING OUT BUT I
DON'T THINK ANY STUDENT WHO IS
EMPOWERED ENOUGH TO MAKE A
DECISION THAT MAY NOT BE THE
Ph.D. ROUTE BUT FEEL LIKE I HAD
A GREAT EXPERIENCE BUT THIS
ISN'T FOR ME BECAUSE I DIDN'T
KNOW WHAT TO EXPECT.
I STILL THINK THAT IS A WIN.
>> THERE'S INTRAMURAL NIH AND
THEY'RE IN THE TIED WITH T32 BUT
SEEM TO HAVE PUT INFLATION AN
ATTRACTIVE THING AND KEEPS
GETTING FILLED UP BY PEOPLE WHO
COME AFTER UNDERGRAD.
T32s HAVE DIFFERENT ROLES AND
CONSTRAINTS AND NOT TETHERING IT
BUT ACHIEVING DIFFERENT GOALS.
>> LET THIS BE A PUBLIC SERVICE
ANNOUNCEMENT TO LET CONGRESS NOU
KNOW THERE SHOULD BE MORE
FUNDING FOR THIS.
I AGREE IT COULD BE EXPAND.
IT'S DIFFICULT TO COMPARE TO
THE PREP PROGRAM AND INTRAMURAL
PROGRAM BECAUSE THEY DON'T HAVE
ANY CONSTRAINTS.
YOU COULD HAVE HAD FIVE YEARS
WORTH OF RESEARCH EXPERIENCE
COME INTO THESE PROGRAMS, HAVE
AN ADDITIONAL EXPERIENCE AND
MOVE ON TO THE NEXT FACE.
AND WE'RE THINKING STRATEGICALLY
A LOT OF TRAINEES FROM UNDER
REPRESENTED PROGRAMS GO TO
SCHOOLS THAT DO NOT HAVE ACCESS
TO RESEARCH.
THEY SHOULD NOT BE LEFT OUT
SIMPLY BECAUSE THEY HAVEN'T HAD
THE OPPORTUNITY TO GET THE
EXPERIENCE IN UNDERGRADUATE.
THIS IS A SPECIAL POOL OF
TRAINEES THAT ARE OFTEN STRUNG
ALONG AND DON'T HAVE REAL
GUIDANCE.
PART OF THE REASON THIS IS TIED
TO THE T32 THEY'RE GETTING
EXPOSURE TO AN ENTIRE PROGRAM
A
AND UNDERSTANDING HOW THE GAME
WORKS AND HOW RESEARCH IS DONE
AND THAT MAKES THE PROGRAM
IMPORTANT AND HOPEFULLY WE CAN
EXPAND IT ONCE WE HAVE DATA TO
SHOW YOU, THAT YES IT WORKED AS
WE HOPED IT WOULD.
IT'S A RISK WORTH TAKING.
>> THE INSTITUTIONS SUPPLYING
THE STUDENTS IN THE PROGRAMS AND
I NOTICED IT'S A MIX OF WELL
HEALED COLLEGES WHO PROBABLY
HAVE SIMPLE RESEARCH GOING ON
AND EVEN UNDER GRADS THAT ARE
PRIZED.
IS THERE EFFORT TO VET THE
INSTITUTIONS PROVIDING THEM AND
ONCE WELL HEALED VERSUS ONES NOT
SO MUCH.
>> THAT'S UP TO THE PROGRAM
DIRECTORS TO SELECT STUDENTS.
SOME INSTITUTIONS FOR INSTANCE
MY ALMA MATER IS KNOWN TO
PRODUCE STUDENTS WHO HAVE ACCESS
TO RESEARCH BUT DON'T HAVE
ACCESS TO GRADUATE PROGRAMS.
EVEN IF THEY'RE ABLE TO DO
RESEARCH, THEY DON'T HAVE THE
EXPOSURE TO A GRADUATE Ph.D.
LEVEL RESEARCH ENVIRONMENT.
THAT'S STILL AN IMPORTANT PIECE
THAT CAN'T BE OVERLOOKED.
JUST LOOKING AT THE GROUP SAYING
THEY COULD DO THEIR Ph.D. THEY
HAVE ACCESS.
BECAUSE IT'S SUCH A NEW PROGRAM
AND THE RESTRICTIONS ARE THERE,
THEY'RE QUITE STRINGENT BUT HAVE
RISEN TO THE OCCASION AND LOOKED
AT WHAT THE RIGHT MIX IS.
WE'RE HAVING NOT QUITE QUARTERLY
BUT TRYING TO MEET WITH THE
P.I.s AND MYSELF AND APPROACH
INSTITUTIONS THAT COULD USE A
LITTLE BIT MORE ATTENTION.
I THINK IT CAN BE A WORK IN
PROGRESS BUT IT'S NOT UP TO ME.
I'M JUST THERE TO GUIDE THEM AND
LET THEM KNOW, NO, NO, NO.
YOU CAN'T GET SOMEONE FROM U.C.
IRVINE.
THAT'S NOT WHAT WE'RE LOOKING
FOR BUT I THINK IN GENERAL
THEY'RE DOING REALLY WELL.
>> A RUN A SUMMER PROGRAM AT
UCLA THAT BRINGS IN STUDENTS
FROM HBCUs AND HISPANIC-SERVING
INSTITUTIONS.
ACTUALLY I DON'T RUN IT ANYMORE
MY COLLEAGUES RUN IT NOW.
MAYBE WE COULD INSTITUTE ADDING
THAT AT THE SAME INSTITUTIONS
AND MAY SAY THE RESEARCH IS FOR
ME AND THEN SELECTED TO THE
PROGRAM.
>> I WAS TALKING WITH MY
COLLEAGUES IN THE OPEN OFFICE
HOW TO BETTER INTEGRATE ACROSS
THE R25 PROGRAMS THAT PROVIDE AN
EIGHT TO TEN-WEEK TEST OF
RESEARCH AND LOUING THEM TO KNOW
THE OPPORTUNITIES EXIST AND
PERHAPS IT'S PART OF AN EXIT
PACKAGE AND FUNNEL FOLKS GOING
INTO THEIR SENIOR YEAR AND HOPE
WE CAN FIND BETTER WAYS TO PUT
TOGETHER THE PROGRAMS WE'RE
ALREADY FUNDING AND ALREADY
INVESTED IN.
>> ALL RIGHT, THANK YOU FOR THE
ROBUST DISCUSSION.
NOW WE'LL PUT UP A SLIDE.
OKAY SO THESE ARE THE OTHER
CONCEPTS.
MOST ARE REISSUED AND THIS IS
THE OPPORTUNITY IF THERE'S
QUESTIONS ON THESE TO GET THEM
ANSWERED.
HEARING NO QUESTIONS, I'LL ASK
FOR A MOTION TO MOVE FORWARD ON
ALL OF THE CONCEPTS THAT HAVE
BEEN PRESENTED ON THE SLIDE OR
DISCUSSED HERE.
TIM HAS MADE A MOTION.
IS THERE A SECOND?
GINA MADE THE SECOND NOW.
I'LL GO TO MY LIST AND CALL OUT
THE NAMED.
AMY.
>> APPROVED.
>> BOB.
>> APPROVE.
>> JEN.
>> APPROVE.
>> JANG.
>> APPROVE.
>> JIM MU.
>> APPROVE.
>> JOHN.
>> APPROVE.
>> LOUISE.
>> APPROVE.
>> HANK.
>> APPROVE.
>> GINA.
>> APPROVE.
>> GEMMINI.
>> APPROVE.
>> JIM.
>> EXCELLENT.
WITH THAT WE'RE AT THE END OF
THE OPEN SESSION.
I'M GOING TO ADJOURN IT.