>> GOOD MORNING, EVERYONE.
WELCOME TO THE 2025 NINDS
NON-PROFIT FORUM, I'M THE DEPUTY
DIRECTOR OF NINDS, PROUDING T
O -- PROUD TO BE HERE AND
HONORED FOR THE 20th GATHERING
OF THE FORUM.
THIS 20 YEARS REFLECTS A
LONGSTANDING COMMITMENT TO
PARTNERSHIP WITH THE COMMUNITY,
AND REFLECTS OUR MISSION, TO DO
BASIC NEUROSCIENCE TO UNDERSTAND
DISEASES AND TO IMPROVE THE
LIVES OF PEOPLE WITH LIVED
EXPERIENCES WITH NEUROLOGICAL
DISORDERS.
IT IS CERTAINLY IMPORTANT TO
RECOGNIZE THE PUBLIC/PRIVATE
PARTNERSHIPS ARE FUNDAMENTAL,
THE AMERICAN BRAIN COALITION WAS
INSTRUMENTAL IN SUPPORTING US
AND GETTING SUPPORT FROM
SPONSORS TO ORGANIZERS OF THE
EVENT.
THE NIH AND NINDS HAS BEEN
THROUGH A LOT OF CHANGE THE LAST
FEW MONTHS, WE'VE LOST A LOT OF
STAFF MEMBERS WHO IN THE PAST
HAVE BEEN KEY IN ORGANIZING THIS
EVENT, MAKING IT POSSIBLE.
SO WE'RE MOST, MOST GRATEFUL TO
ABC FOR BEING HERE ALONG WITH US
AND FOR BRINGING THIS WONDERFUL
COMMUNITY TOGETHER.
WE'RE GOING TO HEAR ABOUT THE
STATE OF THE SCIENCE,
PUBLIC/PRIVATE PARTNERSHIPS AND
STORYTELLING, REALLY ORGANIZED
INTO THREE SESSIONS,
CROSS-CUTTING THEMES AMONG ALL
SO I ENCOURAGE YOU ALL TO THINK
OUTSIDE OF THOSE BOXES AND TALK
AMONGST THE GROUPS IN THE
BREAKS, WHICH WE'VE ORGANIZED
MANY.
THIS IS ALSO A MILESTONE YEAR
FOR NINDS, THE 75th
ANNIVERSARY OF NINDS, SO IT'S
PARTICULARLY SPECIAL TO BE HERE
WITH ALL OF YOU TO TAKE A MOMENT
TO ACKNOWLEDGE AND RECOGNIZE THE
WONDERFUL SCIENCE BEING DONE AND
CELEBRATING THE COMMUNITY AND
BEING HERE TOGETHER.
A FEW ORGANIZATIONAL NOTES, THE
SESSION IS BEING RECORDED, A
LINK TO THE RECORDING WILL BE
SHARED WITH EVERYBODY ON THE
NINDS WEBSITE.
FOR THOSE PARTICIPATING
VIRTUALLY THERE'S A Q&A FEATURE
ON TEAMS THROUGH WHICH YOU CAN
SUBMIT QUESTIONS, MONITORED
THROUGH THE DAY.
FOR THOSE HERE IN PERSON, THERE
ARE STANDING MICROPHONES ABOUT,
WE CAN BRING THEM OVER HANDHELD
IF THAT'S EASIER, CHAT FEATURE
IS OPEN FOR DISCUSSION DURING
THE MEETING, LUNCH WILL BE
PROVIDED COURTESY OF AMERICAN
BRAIN COALITION AND SPONSOR ELI
LILLY.
WE'LL HAVE TWO QUIET ROOMS, LET
THE STAFF AT THE REGISTRATION
KNOW IF YOU NEED HELP FINDING
THOSE OR ORGANIZING TIME IN
THOSE.
THERE'S ROOM TODAY AND BEYOND
TODAY TO JOIN THE ONLINE
CONVERSATION USING THE HASHTAG
#OR NUMBER SIGN FOR THOSE OF A
CERTAIN GENERATION, NINDS75.
WITHOUT FURTHER ADO I'M GOING TO
MOVE TO THE ACTUAL PROGRAM,
WHICH IS GOING TO BE KICKED OFF
BY THE DIRECTOR OF NINDS, DR.
WALTER KOROSHETZ.
WALTER NEEDS NO INTRODUCTION BUT
A LITTLE BIT OF FUN BACKGROUND
ABOUT WALTER, A BROOKLYN NATIVE,
ATTENDED GEORGETOWN UNIVERSITY
HERE WHERE IF YOU ASK HIM, HE
WILL GIVE YOU A TOUR OF THE
COLLEGE CAMPUS, STUDIED AT THE
UNIVERSITY OF CHICAGO MEDICAL
SCHOOL, STARTED RESIDENCY AND
MOVED TO MASS GENERAL WHERE HE
COMPLETED NEUROLOGY RESIDENCY,
CHIEF RESIDENT.
HE SPENT QUITE A LOT OF TIME AS
A BASIC SCIENTIST LOOKING AT
DISEASE MECHANISMS FOCUSING ON
NEUROPHYSIOLOGY, BASIC
ELECTROPHYSIOLOGY, HUNTINGTON'S
DISEASE BUT BRINGING IN ASPECTS
OF SCIENCE RELEVANT TO MANY
DISORDERS.
HE WENT ON TO HAVE A
FOUNDATIONAL ROLE IN THE FIELD
OF STROKE AND NEUROCRITICAL
CARE, A LOT OF CENTERS THAT DO
THAT NOW AS A MATTER OF COURSE
REFLECT WALTER'S EARLY VISION IN
THE FIELD.
CAME TO THE NINDS IN 2007, HAS
BEEN DIRECTOR FOR 10 YEARS, A
LOT OF MILESTONES THIS DAY AND
THIS REFLECTS A DECADE OF WALTER
BEING HERE AS A LEADER OF THE
NEUROSCIENCE COMMUNITY.
WITHOUT FURTHER ADO, WALTER,
HAPPY TO HAVE YOU KICK US OFF.
>> THANKS VERY MUCH.
WELCOME TO THE NON-PROFIT FORUM.
PARTICULARLY OUR KEYNOTE
SPEAKER, CONGRESSWOMAN JENNIFER
WEXTON IS HERE, TALKING TO YOU
IN A BIT.
A SPECIAL DAY FOR US AT NINDS,
AND WE'RE SO GRATEFUL TO THE
AMERICAN BRAIN COALITION FOR
HELPING US IN THIS TOUGH TIME, I
THINK WHEN TIMES GET TOUGH, THE
TOUGH GET TOUGHER.
AND BAND TOGETHER.
THAT'S WHAT HAPPENED.
WE WERE UNABLE TO RUN MEETINGS
BY OURSELVES AFTER THE REDUCTION
IN FORCE BUT AMERICAN BRAIN
COALITION CAME TO THE RESCUE AND
SO WE HAVE A REALLY GREAT
PROGRAM.
ONCE AGAIN, I SAY IT EVERY YEAR,
IT'S GOING TO BE HARD TO BEAT
LAST YEAR, BUT I THINK WE ARE
GOING TO DO IT THIS YEAR AGAIN.
THIS IS PUT TOGETHER BY THE
NON-PROFITS THEMSELVES.
WE HELP AT NIH, BUT IT'S REALLY
FOR THE NON-PROFITS, FOR US TO
LISTEN TO THEM, FOR THEM TO HEAR
THE NEWS FROM US, AND ALSO FOR
THE NON-PROFITS TO WORK TOGETHER
AND KIND OF COME TOGETHER AND
LEARN FROM EACH OTHER, SO THEY
CAN BE MORE SUCCESSFUL GOING TO
THE NEXT YEAR.
I HAVE HERE THE MEMBERS OF
PLANNING COMMITTEE WHO WERE
INVOLVED IN THE NON-PROFIT
FORUM, HELPING US TO SET THE
AGENDA, AND I THINK IT'S GOING
TO BE A REALLY FANTASTIC DAY.
I ALWAYS LEARN A LOT.
LOTS OF GOOD MEMORIES THAT YOU
HEAR AT THIS MEETING THAT STICK
WITH YOU THROUGHOUT THE REST OF
THE YEAR.
NOW I WANT TO HAVE THE PRIVILEGE
OF INTRODUCING MARK RASENICK,
CHAIRMAN OF THE AMERICAN BRAIN
COALITION, AND MARK HAS HAD A
REALLY FANTASTIC CAREER AT THE
UNIVERSITY OF ILLINOIS IN
CHICAGO, WHERE HE IS PROFESSOR
OF BIOPHYSICS, PHYSIOLOGY, AND
PSYCHIATRY.
THAT WAS ALWAYS MY HOPE AS A
YOUNG KID IN COLLEGE BASICALLY,
TO UNDERSTAND HOW BIOPHYSICS
ALLOWS OUR BRAINS TO DO WHAT WE
DO.
I NEVER REALLY QUITE GOT THERE.
NEITHER DID THE REST OF THE
FIELD.
BUT WE'RE MAKING PROGRESS.
BUT MARK HAS BEEN INSTRUMENTAL
IN MANY ASPECTS OF TRYING TO
ADVANCE NEUROSCIENCE OVER HIS
CAREER, HE WAS THE ROBERT WOOD
JOHNSON FELLOW WITH SENATOR
EDWARD KENNEDY FROM
MASSACHUSETTS, MAYBE THAT WAS
HIS START INTO UNDERSTANDING THE
IMPORTANCE OF MESSAGING THE
VALUE OF RESEARCH, NOT ONLY AT
THE CONGRESSIONAL LEVEL BUT AT
THE NON-PROFIT LEVEL.
HE'S PARTICULARLY BEEN INVOLVED
IN PROSECUTING THE IDEA THAT
NEUROSCIENCE IS GLOBAL, AROUND
THE WORLD, AND SO HE HAS FRIENDS
IN EVERY CORNER THAT YOU CAN
IMAGINE.
AND HE'S A REAL MENSCH.
SO I WANT TO INTRODUCE HIM, I'M
SURE YOU'RE GOING TO LEARN SOME
INTERESTING TID-BITS.
BUT JUST TO SAY THAT AS WE
ALWAYS -- I USUALLY INTERRUPT
HIM AT SOME TIME DURING THE TALK
BUT THE MAIN INTERRUPTION IS
THAT NEUROLOGY IS TAKING OVER
PSYCHIATRY.
WE'RE GOING TO UNDERSTAND HOW
THE CIRCUITS OF THE BRAIN WORK,
AND THAT'S REALLY WHAT IS WHAT
PSYCHIATRY IS ALL ABOUT.
ALL CIRCUIT DISORDERS.
MANY OF OUR DISORDERS ARE
CIRCUIT DISORDERS.
AND THROUGH THE BRAIN INITIATIVE
WE HAVE THE OPPORTUNITY NOW TO
SEE THOSE CIRCUITS IN ACTION AND
I THINK IT'S GOING TO BE REALLY
A POWERFUL NEW TOOL AND
KNOWLEDGE GOING FORWARD.
SO, MARK, THANK YOU VERY MUCH.
>> THANK YOU.
THAT'S VERY KIND, WALTER,
ACTUALLY MY INTRODUCTION TO
ADVOCACY AND ACTIVISM STARTED IN
CHICAGO IN 1968.
IN GRAND PARK.
SO, I'M MARK RASENICK, I'M A
PROFESSOR AT THE UNIVERSITY OF
ILLINOIS, CHAIRMAN OF THE
AMERICAN BRAIN COALITION.
I DON'T NEED FIVE MINUTES.
I THOUGHT YOU HAD CUE CARDS.
AND ABC SERVES AS AN UMBRELLA
ORGANIZATION, UNITING THE
NATION'S LEADING PROFESSIONAL
SOCIETIES IN NEUROLOGY,
PSYCHIATRY, AND PSYCHOLOGY,
ALONG WITH DISEASE-SPECIFIC
ADVOCACY GROUPS, RESEARCH
INSTITUTIONS, AND OUR INDUSTRY
PARTNERS.
OUR MISSION IS TO PROMOTE
UNDERSTANDING OF THE BRAIN AND
REDUCE ENGAGEMENT AND REDUCE --
WE FOCUS ON ENGAGEMENT AND
ADVOCACY.
MY OWN RESEARCH IS FOCUSED ON
NEUROBIOLOGY OF DEPRESSION BUT
AS WALTER SAID, IT'S ONE BRAIN.
AND THE ABC IS COMPLETELYING
AGNOSTIC AS TO INDUSTRY,
NEUROLOGY, TRAUMATIC EVENT
ELICITING A BROKEN BRAIN.
WE'RE ALL TOGETHER IN THIS.
SO THAT THESE BARRIERS ARE
REALLY MORE ARTIFICIAL THAN
REAL, AND HOPEFULLY WE CAN
CONTINUE TO WORK TOGETHER TO
MAKE PROGRESS TOWARDS
UNDERSTANDING BRAIN DISEASE AND
ULTIMATELY CURING IT.
WE WORK CLOSELY WITH FEDERAL
AGENCIES LIKE THE NINDS, AND
WHEN WE LEARNED THE FORUM WAS AT
RISK, DUE TO LOGISTICAL
CONSTRAINTS WE STEPPED IN TO
ENSURE THIS VITAL MEETING
CONTINUES.
WE SEND OUR THANKS TO THE FORUM
SPONSORS, PARTICULARLY ELI LILLY
WHO IS GOING TO FEED US LUNCH.
AND JUST YESTERDAY AFTERNOON,
THE AMERICAN BRAIN COALITION
HOSTED A FORUM, WHERE DR.
KOROSHETZ AND DR. KAYE AND A
PANEL SPOKE TO A PACKED ROOM ON
CAPITOL HILL ABOUT NEUROSCIENCE,
BOTH FROM ADVOCACY, BASIC
SCIENCE, AND CLINICAL SCIENCE
AND A TRANSLATIONAL STANDPOINT.
AND IT WAS REALLY -- IT WAS
FANTASTIC.
AND OUTSIDE THIS ROOM, AND ON
THIS SLIDE, IS A SPECIAL IMPACT
BANNER RECOGNIZING THE HUNDREDS
OF ORGANIZATIONS ACROSS THE
BRAIN SPACE, NON-PROFITS,
ACADEMICS, INDUSTRY PARTNERS,
MANY OF WHOM -- ALL OF WHOM HAVE
SPONSORED THE NINDS FORUM, MANY
OF WHOM ARE MEMBERS OF THE
AMERICAN BRAIN COALITION, AND
MANY OF WHOM SHOULD BE.
AND TO KEEP US ENERGIZED FOR THE
DAY AHEAD I'M HAPPY TO TURN IT
OVER TO JENNIFER FRENCH, OUR ABC
BOARD MEMBER AND EXECUTIVE
DIRECTOR OF THE NEURAL TECHED IN
WORK WHO PLAYED A KEY ROLE IN
SHAPING EVERYTHING TODAY.
JEN.
DOES THAT WORK?
>> YOU ALL CAN HEAR ME, RIGHT?
LET'S GIVE APPLAUSE TO CHARLIE,
MAKING ALL OF OUR AUDIO/VISUAL
WORK FOR US, SO WE FIGURED WE'RE
GOING TO BE HAVING PRESENTATIONS
AND PANELS, TALKING ABOUT THINGS
SERIOUS BUT WE -- IT'S
SUMMERTIME, GUYS, WE ALL FEEL
OUTSIDE.
IT'S TIME TO DO SOMETHING A
LITTLE MORE ENGAGING, IF YOU
WILL, TO LET US GET TO KNOW EACH
OTHER.
WHILE CHARLIE BRINGS US UP ON
THE SHARE SCREEN, ALL OF YOU
HAVE YOUR CELL PHONES, RIGHT?
EVEN THOSE ON VIRTUALLY, YOU
HAVE A MOBILE PHONE.
ALL RIGHT.
OKAY.
SO, IT'S VACATION SEASON.
WE WANT TO GET TO KNOW YOU.
IF YOU HAVE A MOBILE PHONE, GO
AHEAD AND YOU CAN SCAN THAT QR
CODE.
OUR QUESTION TO YOU IS WHAT IS
YOUR FAVORITE VACATION SPOT IN
NORTH AMERICA?
FOR THOSE ONLINE, YOU CAN ALSO
SCAN THAT QR CODE OR IF THE SCAN
DOESN'T WORK, YOU CAN PUT IN
MENTI.COM AND ENTER 88033640.
AGAIN, 88033640.
AND WE'RE ALL THINKING ABOUT
VACATIONS THESE DAYS, WHILE IT'S
GETTING NICE AND HOT.
OUR KIDS ARE ON BREAK.
WE'RE THINKING ABOUT WHERE WE'RE
GOING TO GO ON VACATION.
WHERE IS YOUR FAVORITE VACATION
SPOT IN NORTH AMERICA?
WE HAVE CARIBBEAN REPRESENTED ON
THERE.
WE HAVE ALASKA, HAWAII WAY OUT
THERE.
I SEE A COUPLE HAWAIIS.
WE SEE WHAT IS GETTING THE
VOTES.
I'LL GIVE YOU A COUPLE MORE
MINUTES.
WOW, I THINK CALIFORNIA AND
NORTH CAROLINA KIND OF HAVE THAT
MED ATLANTIC/NORTHERN CALIFORNIA
OREGON AREA IN THE COMPETITION.
IS THAT THE GREAT LAKES?
NO, NEW ENGLAND JUST POPPED UP.
LOOK AT THAT!
NOT THAT WE'RE IN COMPETITION
BUT WE'RE LOOKING AT WHERE
EVERYBODY HAS THEIR FAVORITE
VACATION SPOTS.
VOTES ARE STILL COMING IN.
THREE-WAY TIE, WHAT DO YOU
THINK?
FOR ANYBODY IN THE VISITOR
BUREAU, FOR THOSE REGIONS OF THE
COUNTRY, JUST SAYING, YOU MIGHT
WANT TO THINK ABOUT SOMETHING.
BUT, YEAH, LOOK AT SOME OF THESE
PLACES.
PEOPLE HAVE BEEN TO ALASKA,
THERE'S HAWAII OUT THERE.
WE ADOPTED CENTRAL AMERICA.
THAT'S OKAY, SOME OF YOU IN
CENTRAL AMERICA, PARTS OF CUBA,
OH, WOW, THE SOUTH IS STARTING
TO BUILD UP.
I'M NOT OFFICIALLY ON ANY
VACATION BOARD BUT WANTED TO LET
YOU GUYS KNOW WHAT WE'RE
THINKING OF.
A FUN EXERCISE.
WE HOPE YOU'RE NOT THINKING
ABOUT VACATION TODAY WHILE WE'RE
HAVING OUR PROGRAM BUT SOMETHING
TO KEEP IN THE BACK OF YOUR HEAD
WHAT WE'RE THINKING ABOUT AND
SOMETHING TO TALK ABOUT LATER
WHILE WE HAVE LUNCH.
OUR NEXT QUESTION TO YOU IS A
LITTLE MORE RELATED TO WHAT
WE'RE GOING TO BE TALKING ABOUT
TODAY.
A LITTLE OBSCURE.
WHAT FOOD OR DRINK WOULD YOUR
BRAIN BE TODAY?
THINK ABOUT THAT.
WHAT FOOD OR DRINK WOULD YOUR
BRAIN BE TODAY?
BE CREATIVE.
I'M GOING TO GIVE IT A FEW
MINUTES WHILE LETTING RESPONSES
COME N IN.
THIS IS NOT COMPETITION, THIS IS
CREATIVITY.
WE WANT TO KNOW WHAT FOOD OR
DRINK YOUR BRAIN IS TODAY.
WE'RE STARTING TO BUILD UP.
HERE'S THE BIG REVEAL.
IRONIC, COFFEE IS FRONT AND
CENTER.
OKAY, JOHN NGAI FROM THE BRAIN
INITIATIVE, JUST LETTING YOU
KNOW.
IT'S HOT.
ICED COFFEE AND COFFEE GO
TOGETHER.
SOME OF US ARE WATER.
SOMEBODY'S ALREADY THINKING -- A
FEW PEOPLE ARE THINKING ABOUT
5:00.
I SEE A MARGARITA IN THERE.
OATMEAL, WATERMELON.
WOW, YOU GUYS ARE CREATIVE.
WE HAVE A CREATIVE GROUP AMONGST
US.
JUST TO LET YOU KNOW THERE IS --
WE'RE IN THE LIQUID SPACE.
I'M JUST SAYING.
I SEE A LOT OF COFFEE, WATER,
TEA, SOUP, ESPRESSO, SMOOTHEES.
I THINK WE'RE A BIT IN THE
LIQUID SPACE, DON'T YOU THINK?
ALTHOUGH I DO SEE A SLUSHIE IN
THERE, YEAH.
SO, JUST TO GET US THINKING
ABOUT WHAT WE THINK ABOUT OUR
BRAINS AND WHAT WE'RE THINKING
ABOUT TODAY, SO, I WANT YOU TO
THINK ABOUT NOT ONLY WHAT YOUR
BRAIN IS DOING TODAY BUT HOW WE
CAN FEED YOUR BRAIN TODAY AND
WHAT OUR PANELISTS AND OUR
SESSIONS TODAY WILL HELP TO FEED
YOUR BRAIN.
SO THANK YOU SO MUCH FOR
PARTAKING IN OUR FUN EXERCISE
FOR THE DAY.
WE HOPE THE REST OF THE DAY WILL
BE JUST AS FUN.
I'M SURE IT WILL BE.
AT THIS POINT I WANT TO HAND IT
OVER TO DR. WALTER KOROSHETZ.
WALTER?
>> THAT WAS FUN, JEN.
THANKS SO MUCH.
WE'LL HAVE TO DO THAT MORE OFTEN
HERE AT NINDS.
WE HAVE A FUN GROUP, A FUN GROUP
HERE.
I WANTED TO TALK A LITTLE BIT
ABOUT LAST 75 YEARS IN 10
MINUTES.
AND SO, YEAH, THERE'S A LOT TO
SAY.
I THINK THERE ARE SOME TAKEHOME
POINTS THOUGH.
I THINK MAYBE THE FIRST TAKEHOME
POINT IS HOW WE GOT ACTUALLY TO
BECOME NINDS.
AND IT WASN'T A GROUP OF
SCIENTISTS SAYING WE NEED AN
NINDS.
IT WAS MARY LASKER, WHO WAS A
FAMOUS PHILANTHROPIST WHO HAS
CONNECTIONS IN CONGRESS, AND SHE
SAW, YOU KNOW, WHAT STROKE HAS
DONE TO HER FAMILY AND WHAT
NEUROLOGIC DISORDERS HAVE DONE
TO OTHERS, AND SHE THOUGHT WE
NEEDED A NEUROSCIENCE INSTITUTE
AND PUSHED AND PUSHED AND
PUSHED, I'M SURE PEOPLE PUSHED
BACK.
ACTUAL FACT, THERE WAS A BIG
PUSHBACK JUST BEFORE SHE WAS
VERY UNSURE WHETHER IT WAS GOING
TO GET PASSED BECAUSE PEOPLE IN
CONGRESS SAID, WELL, WE ALREADY
HAVE THREE INSTITUTES.
IF WE HAVE A FOURTH, EVERYBODY'S
GOING TO WANT ONE.
WELL, THAT CAME TRUE.
27 INSTITUTES AND CENTERS.
AND THE OTHER FUNNY STORY, PART
OF IT, WAS SHE HAD A FRIEND
WHOSE -- WHO HAD BLINDNESS IN
THE FAMILY, AND SAID, WELL, CAN
YOU GET BLINDNESS ON TOP OF
NINDS, THE NEUROLOGIC INSTITUTE?
SHE SAID, THAT'S A GREAT IDEA,
BUT I HAVE TO GO BACK AND PUSH
IT WITH THE CONGRESS.
SHE CALLED ONE OF THE SENATORS,
THINKING IT WAS GOING TO BE A
WEEKS-LONG PROCESS.
SHE SAID WE WANT TO PUT
BLINDNESS.
HE JUST INTERRUPTED HER AND
SAID, OH, THAT'S A GOOD IDEA.
AND THAT WAS IT.
TURNED OUT HIS MOTHER DEVELOPED
BLINDNESS, SO A LITTLE INSIGHT
INTO HOW CONGRESS WORKS.
IT STILL WORKS THAT WAY.
AND THAT'S NOT SO BAD BECAUSE
THAT'S REALLY A STORY ABOUT HOW
BAD THINGS HAPPEN TO REAL PEOPLE
INFLUENCES DECISION OF OUR
LEGISLATORS TO MOVE, TO DO
SOMETHING, NOT JUST FOR THE
FOLKS SUFFERING NOW BUT PEOPLE
IN THE FUTURE.
AND THAT WAS THE BIRTH OF NINDS.
IT STARTED WITH A LOAN FROM THE
INSTITUTE OF MENTAL HEALTH,
MILLION DOLLAR LOAN, STARTED THE
INSTITUTE, BUT THEY DIDN'T GIVE
IT ANY MONEY.
MONEY CAME, IN THE BEGINNING
MOSTLY SCIENTISTS IN BETHESDA
WORKING, SOME REAL
BREAKTHROUGHS, PEOPLE WHO WORKED
ON THE FIRST, YOU KNOW,
TECHNOLOGY THAT LET THE PET
SCANNING, KEN COLE DEVELOPED THE
PREPARATION OF VOLTAGE CLAMPING,
THE SQUID AXON, WHICH HODGKIN
AND HUXLEY CAME AND WORKED WITH
HIM AND THIS GOT NOBEL PRIZE FOR
FIGURING OUT ACTION POTENTIAL.
ED KAYES, HE CAN TELL THE STORY
WORKING WITH ROSCO BRADY, ONE OF
THE PEOPLE WHO REALLY BENEFITED
FROM BEING IN THE INTRAMURAL
PROGRAM.
I'VE BEEN TOLD HE WENT YEARS
WITHOUT PUBLISHING ANY PAPERS,
WHEN PEOPLE ASKED HIM WHAT'S
GOING ON, WHAT IS TAKING SO LONG
HE SAID I'M TRYING TO FIND A
CURE FOR GAUCHER'S DISEASE.
PEOPLE SAID GO AHEAD, ROSCO.
HE DID.
ED WAS INVOLVED IN MOVING THAT
OUT TO THE PATIENTS.
SO WE HAVE I THINK THROUGHOUT
THE DAY WE'LL HEAR GOOD STORIES
LIKE THAT.
AND WHEN YOU THINK ABOUT NINDS,
HERE YOU'RE IN THE BUILDING
WHERE THE STAFF WORK.
THEY ARE WORKING TO GET GRANTS
OUT TO THE SCIENTISTS AROUND THE
COUNTRY.
THAT'S REALLY THE CORE OF WHAT
WE DO.
NINDS IS NOT JUST THE STAFF
HERE.
NINDS IS THE GRANTEES WHO GET
THE FUNDS AND WORK, PUT THEIR
WHOLE CAREERS INTO TRYING TO GET
ANSWERS TO ADVANCE OUR MISSION,
TO UNDERSTAND THE NERVOUS SYSTEM
AND USE THAT KNOWLEDGE TO REDUCE
THE SUFFERING DUE TO NEUROLOGIC
DISORDERS.
PEOPLE PUT THEIR CAREERS OVER
THE LAST 75 YEARS, LOTS OF
PEOPLE PUT THEIR CAREERS INTO
THIS AND WE MADE REALLY GOOD
PROGRESS.
NOT AS GOOD AS WE'D LIKE BUT
WE'VE MADE GOOD PROGRESS.
AND THE OTHER PART OF THE
ECOSYSTEM OF COURSE IS THE
PEOPLE IN THIS ROOM, THE PEOPLE
WHO REPRESENT IT'S PERSONS
SUFFERING WITH NEUROLOGICAL
DISEASES.
THAT'S WHAT IT'S ALL ABOUT.
AND YOU REPRESENT THEM.
WE'RE TRYING TO HELP GET
SOLUTIONS AND HOPE FOR THOSE
FOLKS, SO TOGETHER WE WORK
TOGETHER AND THAT'S WHERE OUR
POWER COMES FROM, NOT SOME
DIRECTOR SITTING IN OFFICE
DECIDING WHAT TO DO.
IT'S REALLY A JOINT COMMUNITY
EFFORT TO DO THE BEST THINGS
WITH THE RESOURCES THE CONGRESS
GIVES US.
AND THEN THE MOST IMPORTANT
GROUP, NEVER FORGET, ARE THE
PEOPLE WITH DISEASES WHO HAVE
TAKEN PART IN THE RESEARCH OVER
THE YEARS.
THERE MUST BE I THINK WE DO A
TALLY EVERY YEAR, HOW MANY
PEOPLE ARE RECRUITED INTO NINDS
STUDIES, IT'S LIKE 100,000
PEOPLE.
IF YOU GO 75 YEARS, YOU'RE
TALKING MILLIONS OF PEOPLE WHO
HAVE TAKEN PART IN RESEARCH.
AND THEY HAVE DONE IT AT GOOD
COST TO THEM, OFTENTIMES THEY
HAVE TROUBLES GETTING TO A
DOCTOR'S OFFICE.
THEY HAVE TO SIT AND WAIT, YOU
KNOW, GO THROUGH ALL THE
TESTING, THEY DON'T DO IT JUST
ONCE.
IF YOU'RE IN A TRIAL YOU'RE
GETTING FOLLOWED UP EVERY COUPLE
MONTHS OR SO.
LOTS OF EFFORT ON THEIR PART AND
THE FAMILIES WHO TAKE CARE OF
THEM AND HELP THEM GET THROUGH
THE RESEARCH.
SO THAT'S OUR ECOSYSTEM.
THE STAFF, THE SCIENTISTS, THE
PEOPLE WITH DISORDERS, PEOPLE
WHO REPRESENT THOSE PEOPLE WITH
DISORDERS.
AND AS I SAID IN THE BEGINNING,
IF IT'S TOUGH TIMES, WE'RE ALL
GOING TO GET TOUGHER AND WORK
TOGETHER.
NOW, IF YOU THINK ABOUT THE LAST
75 YEARS, I MEAN, YOU KNOW, JUST
WHAT WE JUST SAID WE DIDN'T
ACTUALLY KNOW HOW NEURONS
TRANSMITTED IMPULSES.
AND IT WAS KEN COLE AND HODGKIN
HUXLEY THAT LED TO THOSE
DISCOVERIES.
SO IN THE 75 YEARS WE'VE EXIST
IT'S WE'VE GONE FROM REALLY NOT
REALLY UNDERSTANDING ANYTHING
ABOUT THE BRAIN TO HAVING GOOD
KNOWLEDGE ABOUT CERTAIN
COMPONENTS, BUT STILL IF I HAD
TO GUESS I'D GUESS WE KNOW MAYBE
2% OF HOW THE BRAIN WORKS.
WE HAVE A GREAT DEAL TO LEARN.
THE BRAIN IS A MASSIVE 85
BILLION NEURONS, TRILLIONS OF
CONNECTIONS, AND TO UNDERSTAND
HOW THAT COMPUTER WORKS WE'RE
PICKING AT THE ICEBERG RIGHT
NOW.
TREMENDOUS PROGRESS THROUGH THE
BRAIN INITIATIVE, SEEING HOW THE
CIRCUITS WORK.
IF YOU LOOK AT THE NOBEL PRIZE
AND ADVANCES IN 75 YEARS IN
NEUROSCIENCE PREDOMINANTLY IN
UNDERSTANDING MOLECULAR AND CELL
BIOLOGY ISSUES RELATED TO
NEUROSCIENCE.
I FORGET HOW MANY THERE WERE,
LIKE 38 NOBEL PRIZEs, AND
THREE WERE ON CIRCUIT.
NOW WE HAVE THE TOOLS FROM THE
BRAIN INITIATIVE WHERE IT OPENS
THE DOOR TO SEE HOW THAT
COMPUTER WORKS, AND THAT'S GOING
TO BE REALLY BIG.
THE OTHER THING I THINK IS
COMING THAT'S REALLY IMPORTANT
IS THE USE OF GENOMIC THERAPIES
TO TREAT DISORDERS.
AND WE HAVE BEEN WORKING FOR
YEARS TO DEVELOP A DRUG FOR THIS
DISEASE OR THAT DISEASE, AND A
LOT OF TIMES IT'S -- YOU KNOW,
IT'S BEEN A TOUGH ROAD.
I WORKED IN HUNTINGTON'S DISEASE
FOR A WHILE.
IN 1993 WE HAD A HUGE PARTY IN A
HOTEL, BEAUTIFUL VIEW OF THE
HARBOR IN BOSTON, FOUND THE GENE
FOR HUNTINGTON'S.
RECENTLY IN THE SAME HOSPITAL
FOUND THE GENE FOR DYSMUTATED
ALS MUTATIONS, THESE WERE GOINGS
TO LEAD TO TREATMENTS IN FIVE
YEARS, MAYBE SEVEN, EVERYBODY
WAS SO EXCITED.
NOW IT'S A LONG TIME AND WE
DON'T HAVE TREATMENT BECAUSE
THINGS TURNED OUT TO BE MORE
COMPLICATED ON THE CELL BIOLOGY
SIDE THAN ANYONE HAD IMAGINED.
GENOMIC THERAPIES OFFER
SOMETHING ELSE, TO GET AT THE
ROOT OF THE PROBLEM AND SO THEY
ARE ALSO VERY PRECISE.
GENOMIC THERAPIES CAN BE
TARGETED AT THE CELLS THAT NEED
THEM.
THAT ALLOWS YOU TO MAKE BIG
EFFECT SIZES IN THE CELLS, BUT
NOT CAUSE TROUBLE IN THE REST OF
THE BRAIN OR BODY, MUCH MORE
PRECISE, THAT'S REALLY AMAZING
FEATURE THAT WE'RE LOOKING
FORWARD IN MANY AREAS.
RIGHT NOW MOST THE WORK IS IN
THE RARE DISORDERS, PARTICULARLY
THOSE THAT LEAD TO DEATH OR
SEVERE DISABILITY IN CHILDREN
BECAUSE THERE IS NO ALTERNATIVE.
I THINK ONCE WE UNDERSTAND HOW
TO DELIVER WHAT THE SIDE EFFECTS
ARE, WE'LL BE ABLE TO MOVE INTO
MORE COMMON DISORDERS AND
NON-GENETIC DISORDERS TO
MANIPULATE THE GENOME OF THESE
CELLS TO PROTECT THEM AGAINST
DYING OR MALFUNCTIONING.
I'M REALLY EXCITED ABOUT THE
FUTURE.
IF YOU JUST THINK ABOUT THE PAST
THOUGH, THERE HAVE BEEN SOME
THINGS THAT WE CAN CELEBRATE.
THE FIRST THING IF YOU WANT TO
THINK ABOUT THE BIGGEST PUBLIC
HEALTH IMPACT HAS BEEN IN
STROKE.
MARY LASKER WAS PUSHING FOR
NINDS IN THE BEGINNING BECAUSE
OF STROKE AND PRESIDENT KENNEDY
WHEN HIS DAD HAD A STROKE PUSHED
ON STROKE RESEARCH AS WELL.
BUT STROKE RATES HAVE DROPPED BY
ABOUT 70% SINCE I STARTED IN
MEDICAL SCHOOL.
AND SO IF YOU JUST THINK ABOUT
THAT 70% DROP IN STROKE RATES
MILLIONS OF PEOPLE ESCAPED A
STROKE DUE TO RESEARCH HOW TO
PREVENT A STROKE.
THIS WASN'T SOME MAGIC THAT
HAPPENED.
IT WAS PEOPLE WHO JUST WERE
SLAVING TO FIGURE OUT HOW TO DO
THIS.
ONE OF MY MENTORS, PHIL KESSLER,
FROM OKLAHOMA, HAD A PICK-UP
TRUCK, HE WAS RUNNING THE ATRIAL
FIBRILLATION STUDY USING
ANTI-COAGULATION.
HE WOULD DRIVE HIS TRUCK ALL
OVER NEW ENGLAND, HAD TRIALS
GOING AT DIFFERENT HOSPITALS,
BUT HE KNEW THEY COULDN'T DO IT
ON THEIR OWN AND WOULD GET IN
HIS TRUCK AND KEEP DRIVING
AROUND, COLLECTING SAMPLES AND
DATA FROM PATIENTS.
AND DWHRAWN THAT TRIAL AND OTHD
BENEFITS FOR ATRIAL FIBRILLATION
STROKE.
PEOPLE DO THESE THINGS.
GET IN THEIR TRUCKS, WORK ALL
NIGHT, THEY ARE SO ENGAGED IN
THE VALUE OF WHAT THEY ARE DOING
AND THAT'S WHAT HAS LED TO THESE
ADVANCES.
MULTIPLE SCLEROSIS, YOU KNOW, I
DON'T KNOW HOW MANY DRUGS WE
HAVE NOW, 14, 16 DIFFERENT
DRUGS.
EXACERBATION EMITTING M.S. IS
PRETTY MUCH UNDER CONTROL.
NOW WE LEARNED THAT PROGRESSIVE
M.S. IS THE PROGRESS.
AND WE'VE GOT TO FOCUS MORE ON
THE PROGRESSIVE M.S., AS OPPOSED
TO EXACERBATING REMITTING TYPES
THAT HAVE BEEN SO WELL
CONTROLLED NOW WITH MEDICATION.
WHEN I WAS IN MEDICAL SCHOOL AND
RESIDENCY, WE HAD ONLY STEROIDS,
WE HAD THIS LITTLE JOKE THAT
WHAT WE WERE GOING TO DO WAS GET
RICH BY DEVELOPING A PILL THAT
WOULD BE GOOD FOR ANY NEUROLOGIC
DISEASE.
WE HAD ASPIRIN, DILANTIN,
PREDNISONE ALL IN ONE PILL,
BECAUSE THAT WAS BASICALLY WHAT
WE WERE USING FOR MOST
NEUROLOGIC DISEASES.
AND BUT THE NEURAL PILL NEVER
CAME TO FRUITION, THANK GOD.
WE HAVE MUCH MORE PRECISE
THERAPIES FOR THESE DISORDERS.
THINK ABOUT EPILEPSIES, AGAIN,
WE HAD MAYBE THREE OR FOUR DRUGS
BACK THEN.
NOW THERE'S PROBABLY AGAIN 18
DIFFERENT ANTI-EPILEPTIC AGENTS.
WE STILL HAVE A PROBLEM.
30% OF OF THE PEOPLE STILL HAVE
UNCONTROLLED SEIZURES DESPITE
THE DRUGS, DILANTIN AND TEGRATOL
IS WHAT WE STARTED WITH.
AND WE HAVE THE MIRACLES, SPINAL
MUSCULAR ATROPHY, THERE WAS A
TRIAL TO AFFECT SPLICING OF THE
GOOD GENES, AND, YOU KNOW, A LOT
OF DEBATE WHETHER THIS WAS GOING
TO WORK, WHETHER IT WAS TOO LATE
TO TRY.
WE DECIDED, THE FIRST NATURAL
HISTORY STUDY IN INFANTS, TO
PREPARE FOR THE TRIAL, PEOPLE
THOUGHT THAT, YOU KNOW, YOU'RE
NOT GOING TO BE ABLE TO TREAT
INFANTS.
BUT WE THOUGHT IF WE WERE GOING
TO MAKE A BIG DIFFERENCE YOU
HAVE TO TREAT INFANTS.
THE TRIAL WENT INTO INFANTS, THE
KIND OF TRIAL EVERYBODY KNEW THE
ANSWER BEFORE THE TRIAL WAS OVER
BECAUSE EFFECT SIZE WAS
GIGANTIC.
KIDS THAT WOULD BE DYING ON
VENTILATORS WERE STILL WALKING,
STILL TO THIS DAY DOING PRETTY
WELL.
THAT WAS A MIRACLE OF THE FIRST
GENOMIC THERAPY I THINK FOR US,
AND NOW THERE'S A GENE
REPLACEMENT FOR SMA, SO INSTEAD
OF TREATMENT EVERY COUPLE MONTHS
OF SPINAL FLUID IT'S A ONE-SHOT
INJECTION, THAT'S I THINK BEEN
KIND OF THIS LIGHT PULLING US,
IF WE CAN DO THIS FOR SMA, WHAT
ABOUT ALL THE OTHER NEUROGENETIC
DISEASES?
THERE'S NOT A GOOD EXCUSE WHY WE
CAN'T DO IT FOR MORE AND DO IT
QUICKLY SO I THINK WE'VE MADE A
BIG PUSH HERE AT NINDS TO TRY
AND INVEST IN GENOMIC RESEARCH.
HATS OFF TO AMIR WHO IS HERE
FROM DIVISION OF TRANSLATIONAL
RESEARCH AND FOLKS WORKING WITH
HIM TO GET GENOMIC THERAPIES OUT
THERE.
AND THE DRUG APPROVED FOR
ATAXIA, AMAZING, RON BARTEK IS
HERE, HAS BEEN THE FATHER OF THE
NON-PROFIT FORUM, NOW WE'RE BOTH
GRANDFATHERS.
TOO BAD WE GOT OLDER, BUT, YEAH,
ATAXIA WITH KIDS.
NOW WE HAVE TREATMENT.
SO TIME'S ON OUR SIDE.
A LOT OF TOOLS NOW THAT WE
DIDN'T HAVE FIVE YEARS AGO,
DEFINITELY NOT TEN YEARS AGO,
THAT'S WHAT'S GOING TO REALLY
ADVANCE THE PROGRESS AND I DON'T
THINK THERE'S ANYTHING TO STOP
US.
THE MORE RESOURCE WE HAVE,
FASTER WE GO.
WE'RE GOING TO GO, NO MATTER
WHAT, AND I JUST WANTED TO END
UP BY -- ON THAT POSITIVE NOTE,
THERE'S LOTS OF THINGS YOU COULD
BE NEGATIVE ABOUT THESE DAYS,
BUT THE PEOPLE IN THIS ROOM, WE
STICK TOGETHER, WE CAN DO FEEL
POSITIVE.
SO THAT'S MY STORY.
I'M STICKING TO IT.
AND NOW MY GREAT PLEASURE TO
INTRODUCE OUR KEYNOTE
CONGRESSWOMAN JENNIFER WEXTON;
SERVED VIRGINIA'S 20th
DISTRICTS, A TIRELESS ADVOCATE,
DEDICATION TO BRAIN HEALTH AND
NEUROLOGIC RESEARCH INFORMED BY
HER OWN LIVED EXPERIENCE, LEFT A
LASTING IMPACT.
NOT JUST ON POLICY, ON THE LIVES
OF PATIENTS AND FAMILIES ACROSS
THE COUNTRY, SHE'LL HAVE AN
IMPACT ON YOU TODAY.
DURING TIME IN CONGRESS WAS A
DRIVING FORCE TO BOLSTER
RESEARCH, ENVIRONMENTAL TOXICANT
EXPOSURE, NEURODEGENERATIVE
DISEASE, PARKINSON'S, ATYPICAL
PARKINSON'S, PSP, ALS, MULTIPLE
SCLEROSIS, TO SAY THE REVOLUTION
GENETICS ALLOWED US TO SEE THE
GENETIC DRIVERS OF MANY
DISORDERS BUT ALSO HAS TAUGHT US
THAT THERE'S A CEILING THERE AND
THE MAJORITY OF THE DRIVERS OF
THINGS LIKE ALS AND PARKINSON'S
ARE NOT GENETIC, THEY ARE OUT
THERE IN THE ENVIRONMENT.
WE HAVE TO FIND THEM.
WE STARTED AN OFFICE HERE FOR
THE NEUROEXPOSOME, DAVID JETT
RUNS IT.
AMIR TABIS, TRYING TO GET
RESEARCH GOING TO GRAB ON AND
PROVE WHAT IS REALLY DRIVING
SOME OF THESE WHAT WE CALL
SPORADIC NEURODEGENERATIVE
DISEASE CONDITIONS.
IT'S A REALLY BIG PROBLEM.
WE DON'T HAVE THE GREAT TOOLS WE
NEED TO GET IT BUT WE NEED TO
KEEP WORKING TILL WE DO.
THE CONGRESSWOMAN WAS
INSTRUMENTAL IN PASSAGE OF
NATURAL PARKINSON'S ACT, A
BIPARTISAN ACT TO DEVELOP
COORDINATED WHOLE GOVERNMENT
RESPONSE TO PARKINSON'S DISEASE.
FIRST FEDERAL BILL DEDICATED TO
ENDING PARKINSON'S DISEASE, AND
LANDMARK SUCCESS FOR THE BRAIN
HEALTH COMMUNITY.
IT TOOK A LONG TIME, LOTS OF
THINGS HAPPENED IN THE
GOVERNMENT.
EVENTUALLY NINDS GOT THE
DELEGATION TO RUN THAT PLAN AND
DEVELOP THAT COUNCIL.
WE'RE STILL WORKING THROUGH THE
HHS SYSTEM.
WE HAD GREAT VOLUNTEERS TO WORK
ON THE COUNCIL.
WE PICKED A SLATE BEST WE COULD.
NOW IT'S WITH THE LAWYERS AND
HHS PEOPLE AND HOPEFULLY IT SEES
LIGHT OF DAY BEFORE THE REPORT
IS DUE IN JANUARY.
APOLOGIZE, IT'S BEEN TERRIBLE
TRYING TO GET THINGS GOING.
PLEASE JOIN ME IN WELCOMING THE
CHAMPION FOR THE BRAIN
COMMUNITY, CONGRESSWOMAN
JENNIFER WEXTON.
>> I'M JENNIFER'S HUSBAND,
ANDREW WEXTON.
BRIEF INTRODUCTION, AS MANY OF
YOU MAY KNOW SHE HAS THIS
AMAZING A.I. VOICE THAT
REPLICATES HER ORIGINAL VOICE.
IT'S AMAZING.
AND IT DOESN'T ALWAYS WORK.
WE GOT IT 11 MONTHS AGO,
RECEIVED IT JULY 1 LAST YEAR,
THE FIRST TIME WE USED IT
OUTSIDE THE HOUSE WAS IN THE
OVAL OFFICE WHEN THERE FOR THE
SIGNING OF THE PARKINSON'S BILL.
AND SHE GOT TO SIT AT THE
PRESIDENT'S DESK WHILE SHE
PLAYED IT FOR HIM.
IT WAS QUITE A MOMENT.
ANYWAY, WHEN WE USED IT AT HOME
WHEN SHE TELLS ME TO GET HER
THINGS OR TAKE HER PLACES IT
WORKS FINE.
WHEN SHE TRIES TO UPLOAD A
20-MINUTE SPEECH, IT CAN BE
GLITCHY.
SHE PREVIOUSLY HAD HER
CONGRESSIONAL STAFF HELPING HER
WITH THIS, SO WE'VE BEEN TEXTING
BACK AND FORTH WITH FORMER CHIEF
WHO SENT US A VOICE FILE.
NOT QUITE THE MAIN PROGRAM.
IT'S A TEXT VERSION.
WE'LL PLAY IT.
HOPEFULLY IT WORKS OUT WELL.
APOLOGIES.
>> HELLO.
THANK YOU.
SO GREAT TO BE HERE WITH YOU ALL
THEY NINDS PUBLIC/PRIVATE
NON-PROFIT FORUM TODAY.
I'D LIKE TO EXPRESS MY THANKS TO
ADDRESS YOU ON BEHALF OF
MILLIONS OF AMERICA LIKE ME
BATTLING NEUROLOGICAL DISEASES.
ALMOST TWO YEARS AGO TO THE DAY
I WAS DIAGNOSED WITH PROGRESSIVE
SUPERVISOR NUCLEAR PALSY, PSP,
PARKINSON'S DISEASE ON STEROIDS,
A DESCRIPTOR I CAME UP WITH WHEN
PREPARING 20 COME OUT PUBLICLY.
I WANTED TO DESCRIBE THE
HORRORS, IT APPEARS I NAILED IT
ON THE CRITERIA.
HAVING LIVED WITH PSP FOR SEVEN
YEARS I'M SURE I HIT THE THIRD
ONE ON THE HEAD TOO.
I DON'T RECOMMEND IT.
PSP HAS SEVERELY IMPACTED MY
MOBILITY AND ABILITY TO SPEAK
AND I RELY ON THIS A.I.
RECREATION FOR PUBLIC SPEAKING
AND AT HOME WITH MY HUSBAND AND
SONS, WHEN I WANT TO BE
UNDERSTOOD.
BEFORE MY REVEAL BEGAN THE MOST
I ENGAGED WITH THE IDEA OF
HEALTH WAS PLAYING THE "NEW YORK
TIMES" GAME, I KEPT UP WELL WITH
PHYSICAL HEALTH, RUNNING,
TRAINING FOR RACES, GOING TO THE
GYM IN CONGRESS, TAKING GROUP
FITNESS CLASSES, YOGA AND
STRENGTH TRAINING, EATING
HEALTHY.
I NEVER MISSED MY ANNUAL
PHYSICALS WITH MY PRIMARY CARE
PHYSICIAN, OTHER THAN MAYBE
NEEDING TO LOSE A FEW POUNDS I
WAS IN EXCELLENT HEALTH.
WHAT I HAD NOT ANTICIPATED WERE
INVISIBLE WAYS MY BRAIN WASN'T
HEALTHY AND HOW IT COULD SO
QUICKLY AND DRAMATICALLY
SEVERELY IMPACT MY OVERALL
HEALTH AND WELL-BEING.
WITH THE BENEFIT OF HINDSIGHT I
FIRST BEGAN EXPERIENCING
NOTICEABLE SYMPTOMS IN 2019, I
DIDN'T RECOGNIZE THEM AS
SYMPTOMS OF A PROGRESSIVE
NEUROLOGICAL DISEASE UNTIL MUCH
LATER.
MY TOES WOULD CLENCH AND CURL
INWARD.
IT WAS AND STILL IS MORE
PRONOUNCED IN MY RIGHT FOOT,
WORSE AT NIGHT WHEN I LAY IN BED
TRYING TO FALL ASLEEP.
TWO BANDANNAS I WOULD PUT
BETWEEN MY FEET TO FALL ASLEEP.
I EXPLAINED IT AS NATURAL RESULT
OF WALKING TENS OF THOUSANDS OF
STEPS DAILY IN LESS THAN
SUPPORTIVE SHOES.
AS MONTHS PASSED MY GAIT BECAME
MORE OF A SHUFFLE.
I WOULD HIT THE TOE BOX WITH MY
SHOES ON UNEVEN SIDEWALKS ON
CAPITOL HILL, THERE ARE A LOT OF
THOSE ON CAPITOL HILL.
I WAS USUALLY ABLE TO REGAIN MY
BALANCE I REMEMBER ONE OCCASION
WHEN I TRIPPED ON A BRICK
STICKING UP ONE-HALF TO
THREE-QUARTERS OF AN INCH, I
WENT FLYING, SKINNING MY PALMS
AND KNEES AS I FELL TO THE
GROUND.
I'VE NEVER BEEN GRACEFUL BUT
EXPLAINED THAT AS BEING LAZY OR
TIRED.
IT WAS LATE SUMMER, EARLY FALL
OF 2020.
COVID-19 PANDEMIC WAS RAGING.
I WAS STRESSED KEEPING UP WITH
REPRESENTING CONSTITUENTS AND
ENSURING MY KIDS WERE INVOLVED
WITH VIRTUAL SCHOOL AND
REELECTION.
TWO MORE SYMPTOMS I THOUGHT WERE
DUE TO STRESS BUT LEARNED ARE
SYMPTOMS OF PARKINSON'S, THE
FIRST WAS ONSET OF DYSTONIA IN
MY FEET AND MY HANDS BALLED INTO
FISTS, EVERY NIGHT WHEN I TRIED
TO GO TO SLEEP I WENT FOR A WALK
WITH MY HUSBAND, HE NOTICED MY
FISTS.
HE SAID, OOH, I'M SO ANGRY AND
BALLED HIS HANDS INTO FITS
IMITATING ME AND LAUGHING.
MY ARMS DIDN'T SWING LIKE THEY
USED TO.
UNFORTUNATELY THERE WAS NOTHING
I COULD DO TO STOP MY HANDS FROM
MAKING FISTS WHEN I WENT TO BED.
SECOND AND MORE DISTURBING
SYMPTOM WAS CHANGE IN MY VOICE.
I HAD BEEN DOING TOWN HALLS ON
ZOOM ON TOPICS, I REMEMBER THAT
MY VOICE STARTED WAVERING.
IT WAS AS IF I WERE SCARED OR
FREEZING COLD AND SHIVERING.
IT FREAKED ME OUT.
THAT DOESN'T HELP WHEN YOU'RE
EXPERIENCING AN ISSUE WITH YOUR
VOICE.
MY CHIEF OF STAFF AND
COMMUNICATIONS DIRECTOR WERE --
I'D SAY THEY WERE CONCERNED.
MY VOICE NEVER ACTED UP IN THAT
WAY AGAIN BUT IT WAS SCARY, I
FELT I WAS LOSING CONTROL OF MY
BODY.
AS TIME PASSED I NOTICED I HAD
TROUBLE SPEAKING LOUDLY AND
SLOWLY.
AFTER A TOWN HALL IN A
RETIREMENT COMMUNITY DISTRICT
ONE OF THE RESIDENTS APPROACHED
ME AND ASKED IF I HAD A SPEECH
IMPEDIMENT BECAUSE SHE AND
OTHERS IN THE ROOM SHE SAID HAD
A DIFFICULT TIME UNDERSTANDING
MY FAST TALKING.
AFTER THAT EPISODE I COMPLETED
THE PARKINSON'S SPEECH PROGRAM
BUT SADLY IT WAS NO MATCH FOR
PSP.
I HAD ENOUGH SENSE TO GOOGLE MY
SYMPTOMS, I TYPED WHY DO MY TOES
CLENCH AND CURL INTO THE GOOGLE
SEARCH WINDOW.
THE ANSWER, A COMMON SYMPTOM OF
EARLY ONSET PARKINSON'S DISEASE.
I CAN'T HAVE PARKINSON'S
DISEASE.
THAT'S A DISEASE OLD MEN GET.
THE MORE I READ, THE MORE MY
MALADIES WERE SYMPTOMS OF P.D.
I MADE AN APPOINTMENT WITH MY
PRIMARY CARE DOCTOR.
WHEN SHE ASKED WHAT MADE
IMMEDIATE COME, I WAS PRETTY
SURE I HAD P.D.
SHE DIDN'T TELL ME IT WAS ALL IN
MY HEAD OR UNLIKELY THAT I HAD
P.D.
INSTEAD SHE HAD ME GO THROUGH
BATTERY OF TESTS NOW FAMILIAR,
HUNGRY COCKATOO, JAZZ HAND, THE
WHOLE DRILL, REFERRED ME TO A
NEUROLOGIST.
IT TOOK FOUR MONTHS TO GET AN
INITIAL APPOINTMENT WITH A
NEUROLOGIST, IT WAS VIRTUAL.
MY SECOND APPOINTMENT TOOK PLACE
ABOUT SIX WEEKS AFTER THAT.
WHAT FOLLOWED WAS A CONTINUING
PATTERN OF MY NEUROLOGIST
PRESCRIBING A NEW MEDICATION,
AND MY EXPERIENCING LITTLE TO NO
BENEFIT AND INCREASING DOSAGES
UNTIL I WAS TAKING THE MAXIMUM
RECOMMENDED DOSE.
WE COULD START OVER WITH
MEDICATION.
WE DID THIS WITH THREE BEFORE
SHE BROKE OUT THE GOOD STUFF,
LEFT LEVIDOPA, I TOOK ONE HALF,
IT WAS AS IF THE ANGELS IN
HEAVEN SANG HALLELUIA FOR ME.
I THOUGHT WHY DIDN'T MY DOCTOR
LET ME TRY THIS SOONER?
IT FELT GOOD BECAUSE MY BODY'S
ROBUST RESPONSE VALIDATED BY
DIAGNOSIS, SO I BELIEVED.
AROUND THIS TIME I STARTED
THINKING I COULD BENEFIT FROM A
SUPPORT NETWORK OF PEOPLE WHO
UNDERSTOOD.
THE WIFE OF A COLLEAGUE FROM THE
GENERAL ASSEMBLY WAS DIAGNOSED
NOT LONG BEFORE, I CALLED HIM.
I HAD TOLD MY HUSBAND, CHIEF OF
STAFF ONLY, MY SIBLINGS AND
SPOUSES BUT NOT MY MOTHER OR
KIDS, CONGRESSIONAL LEADERSHIP
OR CONSTITUENTS IN THE FEAR I
WOULD BE OUTED.
MY COLLEAGUE'S WIFE FACE-TIMED
ME THAT NIGHT, SHE PARTICIPATES
IN A SUPPORT GROUP OF HIGH
ACHIEVING WOMEN WITH P.D., MOST
IN NYC, I WASN'T READY TO COME
OUT, SHE ASSEMBLED A CORE GROUP
OF CLOSE FRIENDS IN THE GROUP.
THEY INCLUDED A "NEW YORK TIMES"
BEST-SELLING SHORE, NURSE
PRACTITIONER, A FORMER CHAMPION
TENNIS PLAYER, IT'S A BIG DEAL.
THESE WOMEN WERE ALSO
ACCOMPLISHED.
SO TOGETHER AND KIND AND
WELCOMING, I CAME TO BELIEVE IF
THEY COULD GO ON LIVING AND
WORKING, HAVING FUN AND
LAUGHING, I COULD TOO.
I CAME TO REALIZE I HAD A UNIQUE
PLATFORM FROM WHICH I COULD
HOPEFULLY RAISE AWARENESS ABOUT
WHAT PARKINSON'S DISEASE IS.
AND WHAT IT IS NOT.
I ANNOUNCED MY DIAGNOSIS TO THE
PUBLIC ON APRIL 12, 2023, ON
WORLD PARKINSON'S DISEASE DAY,
IN A VIDEO MESSAGE, I VOWED TO
PUT IN THE WORK TO MANAGE
SYMPTOMS AND DO THE JOB I LOVED
OF REPRESENTING MY COMMUNITY IN
CONGRESS.
I WAS GOING THROUGH PHOTOS ON MY
PHONE AND CAME ACROSS ONE OF A
SQUIRREL ON THE MALL TAKEN TWO
YEARS AGO, JUNE 2023.
I HAD TAKEN WHILE ON A RUN AFTER
6:00 A.M. BEFORE A LONG DAY OF
HEARINGS AND VOTES.
RUNS LIKE THIS WERE A PART OF
THE EXERCISE ROUTINE I UNDERTOOK
TO HELP MANAGE MY SYMPTOMS.
THAT MOMENT STOOD OUT BECAUSE
WHILE I WAS STILL ABLE TO MOVE
AND RUN AT THE TIME, ALMOST
EXACTLY TWO YEARS AGO, I HAD
BEGUN NOTICING THE WAYS MY
MEDICATIONS WEREN'T WORKING AS
WELL AS THEY USED TO AND IT
SEEMED MY SYMPTOMS WERE
PROGRESSING RAPIDLY AND I WAS
FEELING WORSE THAN THE OTHER
WOMEN WITH PARKINSON'S IN THE
SUPPORT GROUP.
I KNEW WELL THE IMPORTANCE OF
LISTENING TO MY BODY AND SOUGHT
OUT A SECOND AND THIRD MILLION
OPINION.
AFTER AN IMPROMPTU CURSORY EXAM
IN MY CAPITOL HILL OFFICE, IT
BECAME CLEAR TELL TALE SIGN OF
MID-BRAIN ATROPHY IN THE SHAPE
OF A HUMMINGBIRDS ON THE MRI
SCAN I WAS BATTLING A MUCH MORE
SEVERE AND FASTER PROGRESSING
FORM, ATYPICAL PARKINSONISM.
PSP HAS NO CURE AND TREATMENT
OPTIONS.
WE HAVE TO BETTER UNDERSTAND,
DIAGNOSE, TREAT, AND I HOPE ONE
DAY CURE AND PREVENT THESE
TERRIBLE DISEASES.
MY JOURNEY TOOK THE COURSE OF
THREE YEARS, MONTHS OF
MEDICATIONS, RULING OUT A
HANDFUL OF OTHER DISEASES,
OPINION AFTER OPINION, TEST
AFTER TEST.
AND EVERY STEP OF THE WAY,
THROUGH NO FAULT OF THE
INCREDIBLE MEDICAL SUPPORT I
RECEIVED, I WAS LEFT WITH FEW
ANSWERS TO THE MOST IMPORTANT
QUESTIONS THAT EVERY PERSON
ASKED WHEN GOING THROUGH AN
ORDEAL LIKE THIS.
WHAT IS WRONG WITH ME?
WHERE DID THIS COME FROM?
WHY ME?
AND HOW CAN I GET BETTER?
IT TOOK ALMOST THREE YEARS TO
FIGURE OUT WHAT WAS WRONG.
I CONSIDER MYSELF A LUCKY ONE
WITH PSPP, WITH CHARACTERISTICS
OF PARKINSON'S, MY BODY STILL
RESPONDS TO LEVIDOPA.
I HAVE MY SUSPICIONS I'LL NEVER
KNOW IF IT CAME FROM SOME ROGUE
GENE OR WAS DUE TO EXPOSURE TO
SOME TOXIN OR SOMETHING ELSE.
I GOT MY HOPES UP ON MORE THAN
ONE OCCASION AFTER HEARING ABOUT
THIS OR THAT MIRACLE DRUG I'VE
SEEN ENOUGH TO KNOW NO
MEDICATION OR TREATMENT REGIMEN
RIGHT NOW IS GOING TO MAKE IT GO
AWAY.
I'VE BEEN ASKING MYSELF THESE
AND MANY OTHER QUESTIONS FOR TWO
YEARS TO NO AVAIL THERE'S ONE
ANSWER WHICH PRESENTED LOUDLY
AND CLEARLY, WE NEED TO DO
BETTER.
MORE THAN 50 MILLION PEOPLE
WORLDWIDE SUFFER FROM
NEURODEGENERATIVE DISEASES LIKE
PARKINSON'S, ALS, ALZHEIMER'S,
M.S., CONDITIONS LIKE MY PSP.
SO MANY QUESTIONS ARE SHARED BY
50 MILLION PEOPLE.
WE NEED TO DO BETTER.
I'VE MADE IT A MISSION OVER TWO
YEARS, I RECOGNIZE UNIQUE
POSITION I HAD AS A MEMBER AND
NOW AS A FORMER MEMBER OF
CONGRESS AND SPOTLIGHT I HAD ON
ME AND WANTED TO MAKE THE MOST
OF IT.
YOU AN UNEXPECTED SIDE EFFECT IS
MY APPEARANCE.
BEFORE I LEFT CONGRESS I HAD A
DOZEN COLLEAGUES TELL ME HOW
BRAVE I WAS.
I DIDN'T UNDERSTAND WHY THAT WAS
THE ADJECTIVE USED TO DESCRIBE
ME.
UNTIL I SAW MY COVER PHOTO ON
THE MARCH ISSUE OF BRAIN AND
"LIFE" MAGAZINE.
MEMBERS OF CONGRESS ARE
NOTORIOUSLY VAIN, MALE
COLLEAGUES COLOR THEIR HAIR
RATHER THAN SHOW GRAYS TO
CONSTITUENTS.
I WASN'T VAIN PER SE, I USED TO
TAKE A LOT OF PRIDE IN MY
APPEARANCE.
THAT WENT OUT THE WINDOW IN 2023
WHEN I LACKED THE DEXTERITY TO
APPLY MAKEUP AND BY 2024 COULD
NO LONGER BLOW OUT MY SPARE.
I'VE LOST 40 POUNDS AND, NO, I
WASN'T TRYING TO.
I WAS PLEASED TO LOSE THE FIRST
10 OR 15 I DIDN'T NEED OR WANT
TO LOSE THE NEXT 20 TO 30, I
FEEL LIKE A SCARECROW.
MY COVER PHOTO IS NOT A BAD
PICTURE BUT I SEE EVERYTHING PSP
HAS DONE TO ME.
THAT'S THE POINT.
I USED TO BE A HOTTIE BUT NOW I
CAN ADD THAT TO THE LIST OF PSP
THINGS ROBBED FROM ME.
I CHAMPIONED AN ACT TO BRING KEY
STAKEHOLDERS TOGETHER WITH
PRIVATE SECTOR PRACTITIONERS AND
CAREGIVERS TO TREAT INCLUDING
PSP.
I MET WITH DEMOCRATIC AND
REPUBLICAN LEADS OF THE BILL, TO
ASK HOW I COULD HELP LEND MY
PERSONAL STORY TO THE BIPARTISAN
EFFORT.
IF YOU'VE BEEN PAYING ATTENTION
TO CONGRESS LATELY, YOU KNOW
THAT WORD, BIPARTISAN, IS A RARE
ONE NOWADAYS.
WE CERTAINLY ENCOUNTERED BUMPS
IN THE ROAD WITH THIS BILL.
THE BILL VERY NEARLY DIED IN
COMMITTEE, AS MANY BILLS DO AS A
CASUALTY OF A PROXY BATTLE
BETWEEN REPUBLICANS AND
DEMOCRATS OVER A CULTURE WAR
ISSUE THAT DIDN'T IMPACT THE
UNDERLYING BILL MUCH.
IT TOOK MULTIPLE AVENUES OF
OUTREACH LIKE TEXTS, LETTERS,
BEFORE WE WERE ABLE TO COME TO
CONSENSUS.
I WAS NOT AFRAID TO PULL OUT ALL
THE SYMPTOMS, PULL ON HEART
STRINGS TO BRING PEOPLE
TOGETHER.
I PLAYED THE "I'M DYING AND THIS
IS PRIORITY" CARD.
COLLEAGUES RESPONDED WITH WORDS
OF COMFORT AND ENCOURAGEMENT AND
MOST IMPORTANTLY WITH COMMITMENT
TO TAKE ACTION.
WHAT I FOUND MOST PRICING WAS
THAT AS I SHARED MY PERSONAL
STRUGGLE IN SUPPORT OF THE
NATIONAL PLAN MY COLLEAGUES
CONFIDED SIMILAR STORIES ABOUT A
RELATIVE, PARENT, FRIENDS, WHO
HAD FACED OR WAS FACING A
SIMILAR BATTLE.
I SHOULD NOT HAVE BEEN SURPRISED
AS PARKINSON'S DISEASE IS NOW
THE FASTEST GROWING
NEURODEGENERATIVE DISEASE IN
AMERICA.
SEEING THE FRUSTRATION OF NOT
HAVING ANSWERS TO THE SAME
QUESTIONS I ASKED MYSELF AFTER
MY DIAGNOSIS MOTIVATED THEM NOT
TO WALK AWAY FROM THE
LIFE-CHANGING LEGISLATION AND
THEY HELPED US PASS IN THE HOUSE
BY 407-9.
FOLLOWING A UNANIMOUS PASSAGE IN
SENATE I JOINED PRESIDENT BIDEN
IN THE OVAL OFFICE FOR THE BILL,
A SPECIAL MOMENT.
I LIVED IN D.C. MY ENTIRE LIFE.
GETTING MY WHITE HOUSE MOMENT
SURROUNDED MY FAMILY MEANT SO
MUCH.
IT WAS NOT THE CIRCUMSTANCES I
ANTICIPATED, RELYING ON WALKER,
USING TEXT-TO-SPEECH TECHNOLOGY,
BUT MY TEAM WAS WORKING ON
DEVELOPING MY NEW A.I., READY
JUST IN TIME FOR THE EVENT.
FIRST TIME I USED THIS WAS TO
SPEAK WITH THE PRESIDENT AT THE
SIGNING.
I SET OUT TO USE MY PLATFORM IN
CONGRESS TO FIGHT FOR BRAIN
HEALTH I STARTED BY READING UP
ON POLICY, HAVING CONVERSATIONS
WITH COLLEAGUES WHO WORKED
CLOSELY ON THESE ISSUES, MEETING
WITH EXPERTS, LOOKING AT WHAT
KIND OF COMMON SENSE LEGISLATION
WE MIGHT GET SUPPORT AROUND.
WHAT I DIDN'T FULLY ANTICIPATE
WAS WHAT MY PLATFORM COULD MEAN
NOT IN TERMS OF VOTES AND POLICY
BUT INVISIBILITY.
I'VE BEEN A LEGISLATOR WHO WOULD
RATHER PUT HER HEAD DOWN AND GET
INTO THE WORK RATHER THAN SPEND
MY TIME IN FRONT OF A CNN
CAMERA.
IN THAT SPIRIT I ENTIRELY
UNDERESTIMATED WHAT WOULD HAPPEN
WHEN USING THE A.M. ON -- THE .
I WAS TRYING TO GET A BILL
PASSED.
A RUN-OF-THE-MILL POST OFFICE
RENAMING BILL, ONE OF MANY THE
HOUSE ADVANCES EACH YEAR.
MY MOTIVATION WAS DOING ANYTHING
TO KEEP DOING MY JOB.
IT TURNED INTO WHAT I HAVE TO
BELIEVE IS ONE OF THE MOST
WATCHED POST OFFICE RENAMING
SPEECHES IN A LONG TIME.
AFTER MAKING HISTORY AS THE
FIRST PERSON TO USE ALTERNATIVE
TEXT ON THE HOUSE FLOOR, I
RECEIVED A GREAT DEAL OF
ATTENTION IN THE PRESS AND MORE
MEANINGFULLY OUTPOURING OF
MESSAGES FROM ADVOCATES AND
THOSE FACING CHALLENGES.
CATCHING ME OFF GUARD AGAIN.
I THINK I DIDN'T ANTICIPATE THAT
ATTENTION BECAUSE I'VE BEEN IN
THE PUBLIC LIGHT AS I SPENT OVER
TWO DECADES IN PUBLIC SERVICE.
MY ENTIRE HEALTH BATTLE WITH PSP
HAS BEEN IN THE PUBLIC EYE.
THE IMPACT ON MY HEALTH AS WELL
AS THE WAYS I'VE WORKED TO ADAPT
IS ON DISPLAY.
MY COLLEAGUES HAVE WATCHED OVER
THE COURSE OF THE YEAR,
STRATEGIC STRIDING ON THE HOUSE
FLOOR TO RELYING ON WALKER, MY
SHARP QUESTIONING MUST NOW BE
DONE THROUGH PREPARED REMARKS.
FOR THOSE FACING CONDITIONS
THESE DISEASES PUSH YOU NOT INTO
THE SPOTLIGHT BUT INTO
ISOLATION.
PSP IS A LONELY DISEASE, ROBBING
MOBILITY, WALK, RUN, TRAVELING
UNDER YOUR OWN POWER AND
COMMUNICATE BY SPEAKING.
I DON'T BEMOAN THE WAYS MY
HEALTH STRUGGLE HAS BEEN PUBLIC,
RATHER I SEE THE PRIVILEGE AND
RECOGNIZE THE OPPORTUNITY.
I HAD THE LUXURY OF TEAM GETTING
ME TO VOTES AND OFFICIAL
COMMITMENTS AND SCHEDULING MY
DAY.
WHILE I NO LONGER HAVE A
CONGRESSIONAL STAFF, MY HUSBAND
AND CAREGIVER, ANDREW, MANAGES
TO GET ME UP AND OUT IN TIME FOR
MY MANY APPOINTMENTS.
I HAVE YEARS OF OLD VOICE
RECORDINGS WHICH MADE IT
POSSIBLE TO DEVELOP A SCARY-GOOD
RE-CREATION OF MY VOICE.
I HAVE THE PLATFORM TO TELL MY
STORY ON MY TERMS, RECOGNIZING
THE PRIVILEGE AND POWER IN THAT.
I'VE TRIED TO MAKE THE MOST OF
IT.
I'VE SHARED THE WAYS PSP
AFFECTED MY DAY-TO-DAY LIFE,
WHAT THE DISEASE IS AND IS NOT.
I'VE CORRECTED COLLEAGUES WHO
SPOKE TO ME LIKE A CHILD,
MISTAKING PHYSICAL DECLINE FOR
MENTAL ONE.
I'VE ENCOURAGED OTHER PATIENTS
TO DO THINGS THAT CAN HELP THEM
ALONG THEIR JOURNEY LIKE
RECORDING THEIR VOICE SO THEY
TOO MIGHT DEVELOP AN A.I. MODEL
AND KEEP THEIR VOICE.
I'VE SHARED MY STORY IN EVERY
WAY I CAN BECAUSE I HOPE IT
MAKES THIS STRUGGLE JUST A
LITTLE LESS LONELY AND
ISOLATING.
I'M GOING TO KEEP UP MY ADVOCACY
FOR THAT REASON.
AFTER I FIRST USED MY
TEXT-TO-SPEECH ON THE HOUSE
FLOOR I RECEIVED A TEXT FROM A
FORMER COLLEAGUE IN THE GENERAL
ASSEMBLY, I KNOW THAT YOU ALWAYS
DO THIS WORK FOR ALL OF US BUT I
WANT YOU TO KNOW MY FRIENDS WHO
USE ASSISTIVE TECHNOLOGY SHOWED
THEIR KIDS YOUR SPEECH, DOING
MORE TO ADVANCE THAN YOU'LL
KNOW.
WHILE YOU'RE KICKING ASS FOR
EVERYONE, YOU JUST CHANGED THE
WORLD FOR MY FAVORITE KIDDOS.
SO PROUD TO KNOW YOU.
READING THOSE WORDS WENT SO MUCH
TO ME.
MY HOPE IS THAT I CAN MAKE THE
MOST GOOD OUT OF THE SITUATION.
I HOPE I CAN CONTINUE TO HELP
BRING GREATER RESOURCES TO
ANSWERING IMPOSSIBLE QUESTIONS
AM TO GIVE PEOPLE GOING THROUGH
THIS COURAGE TO FACE THE
STRUGGLES HEAD ON.
FOR ME THE BEST WAY OUT IS
THROUGH.
I'M GRATEFUL FOR THE WORK SO
MANY HAVE DONE TO BETTER
DIAGNOSE, TREAT, CURE AND
PREVENT THESE CONDITIONS AND
COMFORT YOU BRING TO THOSE
FIGHTING THEM.
TO KNOW THERE ARE BRILLIANT,
DETERMINED, CARE PEOPLE LIKE YOU
MEANS THE WORLD TO SOMEONE LIKE
ME.
SO, THANK YOU.
THANK YOU FOR THE KIND I
INVITATION TO JOIN YOU.
SHARING MY STORY IS EVEN GREATER
THAN THE POWER OF MY VOTE IN
CONGRESS.
THANK YOU FOR ALLOWING ME TO
SHARE THAT STORY TODAY.
>> THANK YOU, CONGRESSWOMAN
WEXTON.
IT MEANS SO MUCH THAT YOU'RE
HERE.
THANK YOU FOR YOUR SERVICE AND
STORE STANDING WITH US AS A
COMMUNITY.
AND FOR SHARING YOUR STORY, YOUR
VOICE, SO PUBLICLY AND TO SO
MUCH EFFECT.
WE TRULY APPRECIATE IT.
BEFORE WE MOVE TO OUR NEXT
SEGMENT I WANT TO LET PEOPLE
KNOW THERE'S WATER AND COFFEE IN
THE BACK SHOULD ANYONE NEED A
LIGHT REFRESHMENT.
THE FIRST PANEL WILL EXPLORE THE
STATE OF RESEARCH LED BY HARVEY
LODISH, WHO JOINS US FROM BOSTON
WHERE HE HAS BEEN A PROFESSOR AT
M.I.T., LEADER IN BIOMEDICAL
SCIENCE, YOU MAY KNOW HIM FROM
THE TEXT BOOKS, MOLECULAR
BIOLOGY OF THE CELL.
IT'S A LONG TEXT BOOK.
MOLECULAR CELL BIOLOGY.
I'M DIGGING INTO THE PAST HERE
BUT ALSO HEAVY.
SO BLAME HARVEY FOR YOUR BOOK
CARRYING.
JOKING ASIDE, HARVEY HAS
EXEMPLIFIED BEING A LIFE-LONG
SCIENTIST, INNOVATOR, MENTOR, A
LIFE-LONG STUDENT.
I MEAN KEEPING AN EYE OPEN
TOWARD THE FUTURE OF SCIENCE,
HOW SCIENCE CAN DRIVE US TO MAKE
CHANGE IN MEDICINE.
WE'VE BEEN PRIVILEGED TO WORK
WITH HARVEY AND THE PANEL.
HOPE YOU'LL TAKE AWAY SOME
MESSAGES OF WHAT WE'VE ACHIEVED
AND WHERE THE FUTURE IS MOVING.
THANKS, HARVEY.
>> HI.
I'M GOING TO LET MY COLLEAGUES
INTRODUCE THEMSELVES.
I THINK THEY KNOW THEMSELVES
BETTER THAN I EVER WILL.
JUST A BIT OF MY OWN BACKGROUND,
BESIDES BEING A PROFESSOR AT
M.I.T., I'VE BEEN IN BIOTECH FOR
WELL OVER 40 YEARS.
HAVING STARTED COMPANIES SUCH AS
GENZIME IN THE 1980s, PROBABLY
THE FIRST COMPANY TO DEVELOP AND
MARKET AND CLINICALLY TEST AND
GET FEDERALLY APPROVED A DRUG
FOR A RARE DISEASE.
IN THAT CASE IT WAS ENZYME
REPLACEMENT, RECOMBINANT FOR
GAUCHER'S DISEASE.
FOR ME IT WAS AN INTELLECTUAL
ACCOMPLISH TO HELP BUILD THIS
RARE DISEASE DRUG.
IT WAS BASED WORK ON WORK FROM
ROSCO BRADY AT NIH, NOT DRIVEN
BY SCIENTISTS BUT A BUSINESS
PERSON WHO BUILT THE COMPANY
AROUND RARE DISEASES.
I LEFT GENZYME IN AFTER ITS
I.P.O.
GAUCHER'S DISEASE IS MOST COMMON
IN ASHKENAZI JEWs, TRANSPIRED
IN 2002 THAT OUR DATA AND HER
HUSBAND WERE CARRIERS.
THE CHILD SHE WAS CARRYING HAD
GAUCHER'S, BY AMNIO, HE WOULD BE
BORN WITH TYPE 1 WHICH DOES NOT
AFFECT CENTRAL NERVOUS SYSTEM.
AND I'M PROUD TO TELL YOU THAT
ANDREW IS NOT ONLY -- HOW OLD IS
HE?
23 AND APPLYING TO MEDICAL
SCHOOL.
AS IT HAPPENED, HE'S HERE IN
WASHINGTON WITH THE GAUCHER IN
WASHINGTON LOBBYING TO CONGRESS
FOR SUPPORT.
THERE MAY BE A GENE FOR THIS
SORT OF THING.
I DON'T KNOW.
BUT WE'RE GOING TO TALK ABOUT
TAKING SCIENCE INTO BUSINESS.
MY COLLEAGUES HAVE A GREAT DEAL
OF EXPERIENCE WITH THIS.
I THINK WE'LL START WITH YOU.
>> EDWARD KAYE, PEDIATRIC
NEUROLOGIST, GENETICIST.
ONE THING YOU'LL HEAR ABOUT
TODAY IS HOW CONNECTED WE ARE.
GENZYME WAS THE FIRST COMPANY I
WORKED WITH, A LOT OF WORK AND
EARLY WORK WAS TO STUDY TYPE
ABOUT GAUCHER DISEASE WORKING
WITH JOHN, AND ROSCO BRADY, IN
THE BRAIN BANK AT NIH WE HAD
SAMPLES, WE HAD A TECHNIQUE TO
MEASURE LIPIDS, I WAS A JUNIOR
FACULTY, JUST STARTING OUT, THAT
WAS VERY HELPFUL.
FOR ME I AM ONE OF THE FEW
PEOPLE THAT TRANSITIONED FROM
ACADEMIA, SPENT 20 YEARS IN
ACADEMIA, SPENT TIME AT MASS
GENERAL, BOSTON CHILDREN'S, AT
CHOP, AND THEN ONE OF MY
FRUSTRATIONS WAS WE WERE MAKING
A LOT OF DIAGNOSES BUT HAD NO
THERAPIES FOR NEUROGENETIC
DISEASES SO I WAS DETERMINED TO
COME UP WITH THERAPIES, WE'RE
WORKING ON GENE THERAPY FOR A
LYSOSOMAL STORAGE DISEASE, IT
HAD TO BE AT A BIOTECH COMPANY,
GENZYME WAS MY FIRST APPROACH.
I MADE THE TRANSITION BACK AND
FORTH, I KNEW WALTER AND ANN.
WE'RE ALL CONNECTED.
ONE OF THE SUCCESSES OF THE
BIOTECH INDUSTRY, AND THAT WAS
TRUE FOR MY CAREER AT GENZYME
AND CONTINUING ON, WE WERE ABLE
TO IDENTIFY GOOD POTENTIAL
MEDICINES OUT OF UNIVERSITIES,
ALL FUNDED BY THE NINDS, THAT
ALLOWED US TO MAKE MEDICINE.
WITHOUT THAT INFRASTRUCTURE,
WITHOUT THAT SCIENTIFIC
INNOVATION, WE COULD HAVE NEVER
MADE THE PROGRESS THAT WE HAVE.
SO I THINK WE HAVE TO REMEMBER
THAT WE ARE SO CONNECTED
TOGETHER, BETWEEN THE BASIC
RESEARCH AND BETWEEN THE
INDUSTRY AND THE PURPOSE I
ALWAYS THOUGHT ABOUT IS
PURPOSES, MAKING PEOPLE BETTER,
MAKING THEM HEALTHY.
WE DO THAT BY USING THE MOST
CURRENT STATE STATE-OF-THE-ART
SCIENCE.
>> CRAIG BLACKSTONE, SCIENTIFIC
DIRECTOR OF MASS GENERAL, THE
MASS GENERAL BRIGHAM SCIENCE
INSTITUTE, PROBABLY THE LARGEST
IN THE WORLD.
IT'S ALL THE ABOUT
COLLABORATION, TRYING TO LINK
ALL THE NEUROSCIENCE TOGETHER,
NOT JUST NEUROLOGY, TO SPEED
CURES, ONE OF THE REAL THINGS IS
MAKING CLINICAL TRIALS MORE
EFFICIENT AND CHEAPER SO WE CAN
TRY MORE THERAPIES.
ANOTHER JOB CHIEF OF MOVEMENT
DISORDERS DIVISION AT MASS
GENERAL, WITH EXCELLENCE IN MANY
AREAS.
IN MANY WE PARTNER WITH YOU.
WE HAVE ONE IN PARKINSON'S, WITH
THE PARKINSON'S FOUNDATION, LEWY
BODY, TOURETTE'S, HOLY HUNTING,
PSP, LEARNING WHAT'S MOST
IMPORTANT, COMING UP WITH NEW
WAYS TO DO TRIALS AND BRING
EXPERTISE TOGETHER IN WAYS THAT
ARE BENEFICIAL TO ALL OF US.
MY CAREER STARTED HERE, I WAS
HERE FOR 20 YEARS AT THE NIH,
UNDER WALTER AND OTHERS, HAD THE
REAL EXPERIENCE TO SEE THE
WONDERFUL WORK IN INTRAMURAL
RESEARCH PROGRAM HERE AND ALL
THE PIONEERING WORK THAT CAN BE
DONE AND HOPEFULLY THAT I CAN
CONTINUE IN THE FUTURE BECAUSE
ABSOLUTELY WONDERFUL AND
SUPPORTIVE PLACE TO BE.
A LOT OF GREAT IDEAS THAT ENDS
UP IN PATIENTS REALLY STARTED
HERE.
>> HI, POOJA KHATRI, PROFESSOR
AND CHAIR OF NEUROLOGY, YALE
UNIVERSITY.
I AM A STROKE SPECIALIST.
I GOT INTO RESEARCH BECAUSE I
WAS EXCITED ABOUT THE FACT THAT
WE HAD TREATMENTS FOR ACUTE
STROKE.
WE JUST FIRST HAD THAT
CLOT-BUSTING DRUG BUT THERE WERE
MORE QUESTIONS TO BE ANSWERED
AND BUILT ON.
I WAS EXCITED ABOUT THE SCIENCE
AROUND CLINICAL TRIALS, A LOT
LIKE CRAIG.
I'M HERE MOSTLY BECAUSE OF MY
ROLE LEADING NIH StrokeNet,
PERHAPS THE LARGEST CLINICAL
TRIAL NETWORK FUNDED BY NINDS,
AND WE'RE CHARGED WITH
HARNESSING THE LEADERSHIP ACROSS
THE COUNTRY TO DEVELOP AND
IMPLEMENT TRIALS IN STROKE
PREVENTION, ACUTE THERAPY,
RECOVERY.
AND ALSO TRAIN THE NEXT
GENERATION.
WE'VE COMPLETED TEN TRIALS, ONE
GOT A CONGRESSIONAL AWARD.
IT EXPANDED ENDOVASCULAR THERAPY
WHERE YOU TAKE A WIRE UP AND
PULL THE CLOT OUT OF THE BRAIN,
VERY EXCISING, AND MANY OTHER
TRIALS THAT HAVE PAVED THE WAY
TO THE NEXT TRIALS, HOPEFULLY
MORE THERAPIES.
WE HAVE 15 ONGOING TRIALS RIGHT
NOW, AND WE'VE ENROLLED OVER
18,000 PATIENTS IN THESE
STUDIES.
BUT BEYOND THE NUMBERS, I THINK
THE THING TO TAKE AWAY AND I
HOPE I CAN TOUCH ON SOME MORE IS
FIRST OF ALL ABOUT BRINGING
GROUPS OF PEOPLE TOGETHER AROUND
THIS COMMUNITY, CREATING AN
INFRASTRUCTURE, A PASSION,
BECAUSE YOU CAN DO GREAT SCIENCE
BUT YOU HAVE TO CONNECT IT TO
THE PEOPLE.
AND IT'S ONLY WHEN EVERYONE
COMES TOGETHER, NO ONE HAS THEIR
NAME BEHIND IT, IT'S A BIG GROUP
ACTIVITY.
I HOPE WE CAN TOUCH ON THAT.
AND THE SCIENCE OF DOING THE
TRIALS THEMSELVES.
WE'VE MADE PROGRESS IN HOW
EFFICIENT THEY ARE, WAYS TO
ANSWER QUESTIONS WITHOUT DOING A
ONE-AND-DONE, BUT WILL IT WORK
FOR THIS GROUP, WHAT IF IT WORKS
FOR A SMALLER GROUP, BUILDING
THAT INTO THE DESIGNS.
THANKS FOR HAVING ME.
I'LL PASS THINGS ON.
>> SO, I'M EVARISTUS NWULIA, I
AM A PROFESSOR OF NEUROSCIENCE
AT HOWARD UNIVERSITY.
I AM A CAREGIVER OF A FORMER
STROKE AND OTHER DISEASES
GRANDPARENTS.
AND CURRENTLY I'M THE DIRECTOR
OF A PROGRAM FOR THE NIH-FUNDED
BLUEPRINT MED TECH PROGRAM SO
THE BLUEPRINT MED TECH PROGRAM
THAT WE SUPPORT IS THE PROGRAM
THAT IS LIKE AN INCUBATOR HUB
THAT BRINGS TOGETHER
NEUROTECHNOLOGIES, DISORDERS,
PROVIDE RESOURCES, PROVIDE A
MENTORSHIP, HELP THEM TO
ACCELERATE DEVELOPMENT TO
COMMERCIAL, SO REALLY EXCITED TO
BE SUPPORTED BY WALTER AND OTHER
NIH INSTITUTES, SOME OF OUR
PROGRAM DIRECTORS ARE HERE.
THIS IS A PROGRAM THAT I REALLY,
YOU KNOW, ENJOY DOING.
I'LL TALK MORE ABOUT THAT AS WE
GO ON.
MY CAREER STARTED AFTER
RESIDENCY TRAINING AT JOHNS
HOPKINS UNIVERSITY, DEPARTMENT
OF PSYCHIATRY.
MY FOCUS THEN IN NEUROSCIENCE
WAS UNDERSTANDING MOLECULAR
MECHANISMS OF BRAIN DISORDERS.
SO I INITIALLY TRAINED IN
GENETICS, NEUROSCIENCE CENTER,
DEVELOPING PLURIPOTENT STEM
CELLS, STUDYING MECHANISMS OF
DISEASES IN ANIMALS.
AS A PHYSICIAN, MY, YOU KNOW,
DISAPPOINTMENT WITH THE PROCESS
IS THE LONG DURATION IT TAKES
FOR US TO GO FROM THIS BENCH TO
EVENTUAL LAB.
I MEAN, TO THE BEDSIDE.
SO TRAGEDY STRUCK MY FAMILY WHEN
MY GRANDPARENTS PASSED AWAY
WITHIN TWO MONTHS, AND SO THAT
WAS A PARADIGM SHIFT FOR ME.
I SHIFTED MY CAREER FROM
NEUROBIOLOGY TO NOW DEVELOPING
NEUROTECHNOLOGY.
AND AS I MOVED ON, DEVELOPED
TECHNOLOGIES, STARTED SMALL
BUSINESSES, MYSELF, I WAS, YOU
KNOW, ABLE TO SEE DISADVANTAGES
SMALL START-UPS, ESPECIALLY FROM
ACADEMIC CENTERS HAVE IN TERMS
OF HAVING THE RESOURCES, HAVING
THE FUNDING, BEING ABLE TO
NAVIGATE, YOU KNOW, THE
COMMERCIALIZATION PROCESS,
REGULATORY PROCESSES, AND WHEN
BLUEPRINT METHOD CAME I FOUND A
WONDERFUL OPPORTUNITY TO USE MY
EXPERIENCE TO ASSIST, PROVIDE
SUPPORT FOR SMALL -- FOR
NEUROTECHNOLOGIES, TO DEVELOP
TECHNOLOGIES THAT CAN REACH THE
HUMAN POPULATIONS.
SO I DESCRIBE MYSELF AS A
NEUROTECHNOLOGYIST, STAKEHOLDER,
CAREGIVING, ALSO A PHYSICIAN.
IT'S AN HONOR TO BE HERE WITH
YOU.
THANK YOU.
>> THANK YOU.
AS YOU'RE HEARING, A REAL
PROBLEM IS TAKING THE RESEARCH
OUT OF OUR ACADEMIC LABS, NIH
LABS, HOSPITAL LABS, INTO
COMMERCIAL PRACTICE SUCH THAT
THE THERAPY CAN BE MANUFACTURED,
MARKETED, AND USED BY PATIENTS
WORLDWIDE.
THERE ARE A COUPLE OF GOOD
EXAMPLES THAT WE'LL TALK ABOUT
IN THIS FIRST SET OF QUESTIONS.
LET ME BEGIN WITH ONE THAT I
THINK REALLY EXEMPLIFIES WHAT
CAN BE DONE, WHICH IS THE
DEVELOPMENT OF THE CYSTIC
FIBROSIS DRUGS.
CF, AS YOU KNOW, AFFECT ONLY
40,000 PEOPLE IN THE UNITED
STATES.
ABOUT 30 YEARS AGO, VERTEX, THE
BIOTECH COMPANY IN BOSTON,
BOUGHT A SMALL BIOTECH IN SAN
DIEGO, AURORA BIOSCIENCE, THAT
HAD DEVELOPED A CELL-BASED ASSAY
WHERE THEY COULD MEASURE THE
FUNCTION OF THE CF PROTEIN, THE
OPENING AND CLOSING OF THE ION
CHANNEL.
AND THEY WERE GOING TO USE IT TO
SCREEN FOR DRUGS FOR CYSTIC
FIBROSIS BUT FELT THE MARKET WAS
BIG ENOUGH AND THEY WERE BACKING
OUT.
IN COMES THE CF FOUNDATION, A
PATIENT-ORGANIZED GROUP OF
PARENTS AND OTHER SUPPORTERS OF
PEOPLE WITH CYSTIC FIBROSIS.
THEY STARTED A FOR-PROFIT
FOUNDATION, WHICH NOW MANY OTHER
NOT FOR PROFITS HAVE MIMICKED,
BUT A FOR-PROFIT FOUNDATION THAT
RAISED AND PUT $150 MILLION INTO
VERTEX TO ENABLE THEM TO DEVELOP
THE FIRST TWO DRUGS THAT WOULD
TREAT A MAJORITY BUT NOT ALL OF
THE CF PATIENTS.
AND THE CF FOUNDATION DID IT
BECAUSE PRIOR TO THIS PROJECT,
THEY HAD SPENT ENORMOUS AMOUNTS
OF MONEY SUPPORTING ACADEMIC
RESEARCH.
THEY HAD VIRTUALLY EVERY PATIENT
GENOTYPE BUT HAD NO DRUG.
SO, THE COMMERCIAL AGREEMENT
ALLOWED FOR A ROYALTY STREAK TO
BE PASSED BACK TO THE FOUNDATION
IN THE SUCCESSFUL EVENT THE
DRUGS ACTUALLY WERE FDA
APPROVED.
SO, THE FOUNDATION STARTED
GETTING A ROYALTY STREAM THAT
THEY SOLD TO A HEDGE FUND CALLED
ROYALTY PHARMA, AND IN RETURN
FOR SELLING THE ROYALTY STREAM
THEY GOT IN CASH OVER $3
BILLION.
THE CF FOUNDATION, I'M A
CONSULTANT FOR THEM, CURRENTLY
IS DEVELOPING GENE THERAPIES NOT
JUST FOR CYSTIC FIBROSIS
ALTHOUGH IT IS THE MAIN FOCUS
BUT FOR OTHER DISEASES THAT
AFFECT THE LUNGS, TRYING TO
UNCOVER SOME OF THE BASIC
BIOLOGY LIKE WHAT ARE THE LUNG
STEM CELLS, SO FORTH.
IT'S REALLY A VERY GOOD EXAMPLE
OF TAKING ACADEMIC DEVELOPMENT,
THE BASIS FOR THE SCREENING, BUT
THE ACTUAL DRUG DEVELOPMENT WAS
DONE BY VERTEX CHEMISTS, IT WAS
NOT DONE IN AN ACADEMIC SETTING.
IT WAS TOO COMPLICATED.
SO IT GIVES YOU ONE EXAMPLE.
ED, YOU'RE ANOTHER EXAMPLE OF
TAKING DRUGS .
IN FACT ALL OF YOU ARE.
WE'LL GO IN ORDER.
GO AHEAD.
>> I WAS VERY FORTUNATE THAT THE
FIRST COMPANY I WORKED FOR WAS
GENZYME, BY HARVEY.
I WAS ABLE TO WORK ON FIVE
DIFFERENT DRUGS, ALL FOR
NEUROGENETIC DISEASES, PRIMARILY
IN LYSOSOMAL STORAGE, DEVELOPING
NEW THERAPIES.
ALL CAME FROM EITHER FROM THE
NIH, FROM MOUNT SINAI, FROM DUKE
UNIVERSITY.
BUT THE UNIVERSITIES THEMSELVES
WERE NOT ABLE, REALLY, TO MAKE A
PRODUCT.
AND TO TAKE IT TO THE NEXT
LEVEL.
MUCH TOO COMPLICATED.
THE MOST RECENT ONE I'VE BEEN
FOCUSED ON AT STOKE THERAPEUTICS
IS AN IDEA THAT CAME OUT OF COLD
SPRINGS HARBOR, ALSO WHERE
WALTER TALKED ABOUT, I GOT TO
KNOW ADRIAN KRAMER QUITE WELL,
HAD A POST DOC THAT BY CHANGING
SPLICING IN THE PRE-MESSENGER
RNA YOU COULD INCREASE AMOUNT OF
FULL LENGTH MESSENGER RNA,
INCREASING PROTEIN.
COULD WE USE THIS AS A PLATFORM
TO INCREASE THE MISSING
PROTEINS?
SO WE WENT AFTER A LOT OF
DISEASES THAT WERE DOMINANT
DISEASES, DIFFICULT TO GO AFTER,
YOU'RE ONLY MISSING 50% OF
PROTEIN BUT THAT CAUSES DISEASE.
SO THE QUESTION CAN YOU BRING
THE UP FROM 50 TO 100%?
SO, THE FIRST YEAR THE COMPANY
STARTED WAS ACTUALLY IN COLD
SPRINGS HARBOR TRYING TO PROVE
DOES THIS MECHANISM WORK.
TURNS OUT IT DOES.
WE'RE IN A PHASE 3 PROGRAM FOR
THE SYNDROME, THIS IS REALLY THE
FIRST TIME WHERE SOMEONE WAS
TRYING TO ADDRESS THE UNDERLYING
GENETIC CAUSE EPILEPSY, WITH A
LOT OF SIDE EFFECTS, YOU ALMOST
CREATE ANOTHER DISEASE.
WE HEARD THIS FROM FAMILIES,
MOTHERS, THEY ARE SLEEPING 18
HOURS A DAY, THAT'S WORSE THAN
THE SEIZURES.
CAN WE ADDRESS UNDERLYING
GENETIC CAUSE?
WE HEARD THIS FROM OTHER GROUPS,
TEN YEARS AGO IF YOU WOULD HAVE
TOLD ME AS A PEDIATRIC
NEUROLOGIST WE COULD IMPROV
DEVELOPMENT OF CHILDREN WHO HAVE
COGNITIVE AND BEHAVIORAL
PROBLEMS I WOULD HAVE THOUGHT IT
WAS IRREVERSIBLE, THAT SHIP
SAILED.
WHAT WE FOUND BY DOING THE
RESEARCH, FOR ANGELMAN'S
SYNDROME AND OTHERS, YOU CAN
ACTUALLY MAKE CHANGES, SEEING
90% IMPROVEMENT IN SEIZURE
CONTROL, IMPROVEMENTS IN
COGNITION AND BEHAVIOR THAT JUST
BLEW US AWAY.
WE DIDN'T THINK WE COULD
ACTUALLY GET THEM BETTER.
WE WERE SEEING IMPROVEMENT EVEN
IN OLDER ADOLESCENTS.
THE IDEA THAT NOW WE'RE AT A
POINT WITH USING ALL OF OUR
MOLECULAR GENETIC INFORMATION
WE'RE ABLE TO SPECIFICALLY MAKE
DRUGS THAT REALLY ARE
DISEASE-ALTERING FOR PATIENTS,
NOT JUST TREATING ONE SYNDROME
BUT THE ENTIRE PATIENT.
WE'RE AT SUCH AN EXCITING TIME.
ONE OF THE REASONS WHY IT'S SO
IMPORTANT TO CONTINUE THE NIH
FUNDING IS THAT IF WE WANT TO
CONTINUE TO MAKE ADVANCES, WE
NEED THAT BASIC INFRASTRUCTURE
OF SCIENCE IN ORDER TO MAKE THE
NEXT GENERATION OF MEDICINES AND
TRAIN THE NEXT GENERATION OF
SCIENTISTS.
THAT'S WHERE THIS NETWORK OF
PEOPLE IS SO CRITICAL, COMING
FROM THE PATIENT GROUPS, THE
ONES BEHIND THIS, SAYING WE NEED
THE THERAPY.
THIS IS WHAT'S WRONG, THIS IS
WHAT YOU NEED TO MAKE.
IT'S THE SCIENTISTS WHO
UNDERSTAND THE MECHANISM OF THE
DISEASE.
THEN IT'S THE DRUG DEVELOPERS
THAT MAKE THE PRODUCT AND THEY
HAVE TO WORK WITH THE FDA TO GET
IT APPROVED.
SO, IT IS THIS NETWORK OF
PEOPLE, THE ONLY WAY WE CAN COME
UP WITH BETTER THERAPIES FOR
PEOPLE.
>> BEFORE WE GO ON, ED, YOU
MENTIONED TWO USES OF
OLIGONUCLEOTIDES TO AEXPECT
SPLICING, SPINAL MUSCULAR DRUG
ATROPHY, YOU COME INTO A GENERIC
TECHNOLOGY THAT COULD BE USED
QUICKER, OR MORE RAPIDLY TO
TREAT OTHER DISEASES WE'RE
TALKING, SO EVERYONE
UNDERSTANDS, THAT'S A
HAPLOINSUFFICIENT GENETIC
DISORDER, ONE COPY OF THE GENE
IS INACTIVE.
SO 50% OF THE NORMAL AMOUNT OF
THE PROTEIN, ION CHANNEL, IS
INSUFFICIENT.
WHAT YOU CAN DO WITH SPLICING
REMARKABLY IS INCREASE THE
AMOUNT OF THAT MESSAGE TO MAKE
ENOUGH PROTEIN THAT YOU CAN
APPARENTLY REVERSE THE DISEASE.
IS THIS A GENERIC TECHNOLOGY?
>> YEAH, I THINK IT COULD BE.
I THINK WALTER MENTIONED ABOUT
THE TREATMENT OF SPINAL MUSCULAR
ATROPHY, DIFFERENT BECAUSE WE
WERE FORTUNATE, WE HAD A SECOND
COPY OF THE GENE, THAT ALMOST
NEVER HAPPENS.
SMN 2 WOULD SPLICE OUT EXON 7,
80% WASN'T WORKING.
WHAT BIOGEN AND ADRIAN KRAMER
FIGURED OUT IF YOU ENHANCE THE
INCLUSION OF EXON AND SMN 2
PROTEIN YOU REPAIR THE PROTEIN
AND JUST ABOUT CURE THE DISEASE.
WHAT WE LEARNED IN EXON
SKIPPING, WE IF THE FIRST
THERAPY FOR DUCHENNE, IF YOU
SKIM OUT ONE EXON YOU MIGHT BE
ABLE TO PUT IT BACK TOGETHER
AGAIN.
THE GENE OF COURSE HAS TO BE
READ, YOU HAVE TO TAKE IT FROM A
DNA TO RNA, THEN MAKE A PROTEIN
OUT OF IT.
IF IT'S NOT LINED UP, YOU CAN'T
READ IT ALL THE WAY THROUGH AND
YOU CAN'T MAKE A PROTEIN.
IF YOU CAN SPLICE OUT NORMAL
SECTION AND PUT IT BACK TOGETHER
THAT COULD WORK.
SO I THINK WHAT WE NOW KNOW AND
NOW THERE IS A LOT OF WORK
LOOKING AT THE UPSTREAM AND
DOWNSTREAM PART OF RNA, ACTUALLY
INCREASING TRANSLATION OF
PROTEIN.
WE HAVE ABILITY POTENTIALLY OF
INCREASING PROTEINS THAT COULD
BE IMPORTANT.
ONE WE'RE WORKING ON FOR AN EYE
DISEASE, WHERE WE'RE REPLACING A
PROTEIN, MITOCHONDRIAL PROTEIN,
BUT ONE OF THE THINGS WE FOUND
OUT IS THAT BY REPLACING THIS
PROTEIN EVEN IN NORMAL CELLS YOU
CAN INCREASE AMOUNT OF ENERGY
CELL HAS, COULD BE USED FOR
OTHER DISEASES, WE TALKED ABOUT
PARKINSON'S, ALZHEIMER'S, THE
CELL DIES, RUNS OUT OF ENERGY.
I THINK WHAT WE'RE SEEING NOW IS
PEOPLE ARE USING SOME RNA
TECHNOLOGY TO GO AFTER A LOT OF
LET'S SAY RARE DISEASES WHERE
YOU HAVE A HANDFUL OF PATIENTS,
AND TECHNOLOGY USED TO FIX THAT
DISEASE.
I THINK THERE'S GOING TO BE MORE
AND MORE OF THAT.
THE CHALLENGE WE'RE GOING TO
HAVE, HOW DO WE ACTUALLY PAY FOR
IT, BECAUSE EACH -- WE SAW THIS
AT MY OLD INSTITUTION AT CHOP
THEY TREATED A CHILD WITH A
DISEASE, A DEATH SENTENCE, THEY
WERE ABLE TO FIX THAT MUTATION,
IT WAS GREAT.
BUT I'M SURE IT COST SEVERAL
MILLION DOLLARS TO TREAT ONE
PATIENT.
SO YOU CAN'T DO THAT FOR
EVERYONE.
SO WE HAVE FIGURE OUT HOW DO WE
USE SOME OF THIS NEW TECHNOLOGY
TO TREAT A LOT OF DIFFERENT
PATIENTS ESPECIALLY WITH RARE
DISEASES IN A WAY THAT'S COST
EFFECTIVE THAT WE CAN AFFORD.
WE HAVEN'T QUITE FIGURED THAT
OUT.
>> VERY GOOD.
CRAIG, YOU GOT -- ALL THREE OF
YOU HAVE DIFFERENT PERSPECTIVES.
GIVE US AN EXAMPLE OF ONE THAT
WORKS.
>> THAT'S HARDER THAN ONES THAT
DON'T WORK.
I CAN TELL YOU CERTAINLY THERE'S
A RARE DISEASE THAT I WORK ON, I
WILL BRING IT UP, A LOT OF
FOUNDATIONS RUN INTO THIS
PROBLEM.
ONE OF MY SPECIALTIES IS
HEREDITARY SPASTIC PARAPLEGIA,
THERE'S 100 FORMS OF IT.
A LOT OF PATIENTS COME AND HAVE
THEIR OWN FOUNDATION, SO HOW DO
WE WORK WITH THE GROUP TO MOVE
THINGS FORWARD FOR EVERYONE?
I CAN GIVE EXAMPLES.
ONE THING THAT SPASTIC
PARAPLEGIC FOUNDATION, ORGANIZED
TO HAVE EXPERTISE IN CERTAIN
AREAS, WE NEED TO UNDERSTAND
WHAT DISEASES LOOK LIKE TO KNOW
IF OUR TREATMENTS ARE WORKING.
WE'VE BEEN DOING THAT.
BUT AT THE SAME TIME SOME RARER
FORMS OF THESE HUNDRED, THERE'S
GENE THERAPY.
A CHILD RECEIVED THERAPY,
RECESSIVE, THE DAD CREATED A
COMPANY SO OTHERS COULD GET THAT
ADVANTAGE.
WE HAVE TO MOVE THIS PROCESS
FORWARD QUICKER.
THAT'S ONE EXAMPLE WHERE IT'S
WORKED.
ANOTHER ONE WITH CHILDREN I TAKE
CARE OF, WORKING WITH THE
FOUNDATION, ANTISENSE
OLIGONUCLEOTIDES, ASOs, WITH
CHILDREN.
EVEN ONCE WE GET IT TO WORK HOW
DO WE INCREASE THE SCALE AND
BENEFIT TO MORE PATIENTS?
FUNDING IS PART BUT A LOT OF
CREATIVITY IN HOW WE MOVE FROM
SCIENCE TO CLINICAL APPLICATION
IS IMPORTANT TOO.
THERE'S LOTS OF WAYS FOR
DIFFERENT TYPES OF DISEASES.
THOSE ARE JUST TWO EXAMPLES IN
THIS BROAD GROUP OF HEREDITARY
SPASTIC PARAPLEGIAS.
AGAIN, AS A CLOSING POINT,
REALLY THE PATIENT IS THE TRUE
DRIVER.
THEY TAUGHT US ABOUT THE
DISEASE.
THEY HELPED US MAKE THE RIGHT
CONNECTIONS AND HAVE BEEN
CRITICAL IN MOVING FROM BASIC
SCIENCE TO ACTUAL TREATMENTS FOR
PATIENTS.
>> THANK YOU.
POOJA, YOU HAD EXPERIENCE IN A
DIFFERENT AREA.
>> YEAH, THOUGH SOME PRINCIPLES
APPLY REGARDLESS OF THE DISEASE.
I'LL TELL YOU ABOUT KIND OF
THREE PATHWAYS THAT STAND OUT TO
ME WHEN THINKING ABOUT TAKING
SCIENCE AND BRINGING IT TO THE
PATIENT.
THE FIRST ONE IS THE SUPPORT
FROM NINDS TO ACTUALLY -- YOU'VE
DONE THE SCIENCE.
HERE IS THE EXAMPLE I'LL GIVE.
MANY OF YOU HAVE HERDS OF tPA,
A CLOT BUSTING DRUG, IF GIVEN IN
A FEW HOURS YOU CAN MAKE A
SIGNIFICANT IMPACT AND REVERSE
STROKE FOR PEOPLE.
I GOT MY CHANCE TO GET MY PUBLIC
HEALTH MESSAGE IN, IF YOU DIDN'T
KNOW THAT.
THERE'S THE OPPORTUNITY TO MAKE
BETTER DRUGS, SO ONE MIGHT HAVE
CLOSE COLLEAGUES, GUY REID, DEAN
AND CARDIOLOGIST, UNIVERSITY OF
ARIZONA, DEVELOPED WITH FUNDING
A NEW MONOCLONAL ANTIBODY,
INHIBITS INHIBITOR OF OUR
BODY'S tPA.
WE MAKE SMALL AMOUNTS.
DEVELOPED THAT THROUGH NIH
FUNDING, A SMALL BUSINESS GRANT
THROUGH NINDS.
PUT TOGETHER A COMPANY, PUT
TOGETHER THE MANUFACTURING, AND
THEN GAVE ME A CALL AND SAID
HEY, I WANT TO TEST THIS IN
STROKE PATIENTS NOW, MY DREAM.
AND WE STARTED DEVELOPING A
CLINICAL TRIAL DESIGN AND WE
CAME UP WITH ONE THAT WE HAD A
LOT OF FUN CREATING WITH SOME
NOVEL ELEMENTS TO MAKE IT
EFFICIENT.
WE'RE CURRENTLY DOING THE DOSE
FINDING STUDY RIGHT NOW.
THAT'S AN EXAMPLE OF TAKING THE
NIH PATHWAY FROM SCIENCE TO
CLINICAL TRIAL.
StrokeNet IS ONE EXAMPLE OF
A NETWORK.
YOU MAY HAVE HEARD OF NEURO
NEXT, IN AREAS OTHER THAN
STROKE.
GOING BACK TO StrokeNet,
WE -- BECAUSE WE HAVE THIS
ROBUST INFRASTRUCTURE WE HAD THE
OPPORTUNITY TO CREATE WHAT'S
CALLED A PLATFORM TRIAL.
THE IDEA WITH THE PLATFORM TRIAL
IS IT'S ONE MASTER PROTOCOL, AND
YOU ARE ABLE TO HAVE ONE GOAL,
OUR GOAL IS TO IMPROVE OUTCOMES
WITH PATIENTS WITH VERY LARGE
STROKES.
AND THEN YOU'RE ABLE TO BRING
THERAPIES IN AND OUT OF THAT
SAME PROTOCOL, SO YOU'RE NOT
WRITING A NEW TRIAL EVERY TIME.
IT'S ONE PLATFORM THAT'S
ENDURING.
WHAT'S EXCITING IS NINDS HAS
ENERGIZED THIS BECAUSE THEY SAID
WE'RE NOT GOING TO ANSWER ALL
THESE QUESTIONS YOU GUYS HAVE,
WE HAVE MANY OTHER DISEASES TO
STUDY.
WE NEED SOMETHING MORE
EFFICIENT..
IT WORKS MORE EFFICIENTLY AND
TURNS OUT A LOT OF COMPANIES ARE
NOW KNOCKING ON OUR DOOR TO RUN
THEIR TRIALS THROUGH THIS
PLATFORM.
I THINK THAT'S ANOTHER EXAMPLE.
AND MY UNDERSTANDING AND OTHERS
MAY KNOW MORE HERE BUT NEURONEX
HAS OPPORTUNITIES FOR INDUSTRY
ENGAGEMENT.
AND THERAPEUTIC DEVELOPMENT,
ANOTHER INITIATIVE, CLINICAL
TRIALS IN RODENTS, THE IDEA HAS
BEEN THAT, YEAH, WE OPEN
ARTERIES FOR PEOPLE WITH STROKE
BUT THERE ARE OTHER MEDICATIONS
THAT CAN PROTECT THE BRAIN AND
KEEP IT FROM DYING WHILE WE'RE
WORKING ON OPENING UP THE
ARTERIES BUT THERE WERE A SLEW
OF THEM.
THEY CREATED A STRUCTURE
LITERALLY RANDOMIZED TRIALS OF
RODENTS, TRYING AGENTS, FOUND A
WINNER, NOW PUTTING THAT WINNER
IN OUR PLATFORM, ANOTHER
EFFICIENT WAY OF NOT HAVING
SPENT SO MANY YEARS AND SO MANY
TENS OF MILLIONS OF DOLLARS
THROUGH EACH ONE OF THOSE
AGENTS.
I'LL STOP THERE AND PASS THINGS
ON.
>> SO, I WAS GOING TO INTRODUCE
YOU -- NO, NO, NO, IT'S FINE.
IT'S OFTEN SAID THAT MEDICAL
DEVICES ARE EASIER TO GET TO
MARKET, FASTER TO DEVELOP.
BUT GO AHEAD.
>> WELL, SOME OF IT IS TRUE.
REGULATORY PATHWAYS PEOPLE WHO
HAVE EXPERIENCED IT CAN TELL
YOU, YEAH, REGULATORY PATHWAYS
TO GET DRUGS TO MARKET IS MUCH
LONGER.
BUT ALSO DEPENDS ON WHAT DEVICES
YOU'RE TALKING ABOUT, HOW
INVASIVE THE DEVICES ARE.
SO, MEDICAL DEVICES CAN BE A WAY
OF DELIVERING DRUGS, THEY COULD
BE CHEMICAL DEVICES, THEY COULD
BE HARDWARES.
AND, YOU KNOW, THEY ALSO, YOU
KNOW, HAVE -- YOU KNOW, PLAY A
ROLE IN DELIVERING GENETIC
THERAPY AND SO FORTH TO THE
SITES THAT YOU WANT TO TARGET.
EXAMPLE IS THE BRAIN.
ONE OF THE BIG HURDLES, WE HAVE
HAD OVER THE YEARS, DEVELOPMENT
OF CRISPR TECHNOLOGY, AND ALL
THE OTHER WAYS TO MANIPULATE,
ALTER, YOU KNOW, GENES IS
GETTING THEM TO THE BRAIN.
SPECIFIC REGIONS OF THE BRAIN.
AND SO DEVICES, DEVELOPING
GENETIC THERAPIES, YOU KNOW, CAN
INTERWOVEN AT TIMES.
BUT, YOU KNOW, THEN YOU HAVE TO
DEAL WITH THE BOTTLENECKS WITH
THE DEVELOPMENT OF THE GENETIC
THERAPY AND ALSO BOTTLENECKS OF
THE MEDICAL DEVICE.
SO IT'S -- AND IT'S NOT TRIVIAL
THE NIH, YOU KNOW, WHICH WE JUST
TALKED ABOUT IS ONE AVENUE THAT
HAS DE-RISKING A LOT OF THESE
EARLY CHALLENGES.
AND I CAN TALK ABOUT A
BLUEPRINT, MedTech, THERE'S
ALSO THE BLUEPRINT NEURAL TECH
PATHWAYS ALSO.
WE ARE INTERESTED IN THE
COMMERCIAL AVAILABILITY OF THE
PRODUCTS.
BUT WE ALSO KNOW THAT INVESTORS
WANT A DRUG OR INTERVENTION THAT
HAS A LARGE MARKET.
SO THE NIH HAS A PANEL OF
EVALUATORS, THE NEW
INTERVENTIONS.
WE ALSO LOOK AT THE DISEASE,
UNMET NEEDS, AND WE CONSIDER THE
FACT THAT SCHOLARS OR INNOVATORS
MAY ENCOUNTER CHALLENGES IN
INVESTMENTS SO WE CONSIDER THE
FACT THAT THERE IS A SMALL, YOU
KNOW, THIS IS A SMALL DISEASE
POPULATION, THEY MAY NOT BE ABLE
TO GENERATE FUNDS, WE DO SUPPORT
THEM TO DE-RISK SOME OF THE
PROBLEMS TO HELP, YOU KNOW,
DRIVE THIS TECHNOLOGY OR
TREATMENT TO THE MARKET.
SO THAT'S ONE AVENUE THAT PEOPLE
CAN, YOU KNOW, SERIOUSLY
CONSIDER.
BACK TO YOUR QUESTION, YOU'RE
RIGHT, THE GENERAL CONSENSUS IS
THAT, YOU KNOW, IT TAKES MEDICAL
DEVICES SHORTER TO GET TO THE
MARKET BUT MOST DEVICES THAT WE
SEE A REALLY COMBINATION.
YOU KNOW, SO COMBINATION OF DRUG
AND SO FORTH.
SO IT'S REALLY HARD, IN TERMS OF
BRAIN, TO AVOID, YOU KNOW, HOW
DO YOU GET THIS TREATMENT TO
SPECIFIC PART OF THE BRAIN.
>> LET ME TURN TO A RELATED BUT
DIFFERENT TOPIC WHICH IS PATIENT
ADVOCACY.
SO A LOT OF YOU ARE FROM THESE
GROUPS, AND YOU WANT TO GET
PHARMACEUTICAL COMPANIES AND
ACADEMIC LABS INTERESTED IN
STUDYING YOUR DISEASE.
HOW TO GO ABOUT IT?
ANY OF YOU WANT TO GIVE IT A
TRY?
OKAY.
>> WE WORK WITH MANY OF YOU.
ONE OF THE MOST EFFECTIVE WAYS
IS OBVIOUSLY EARLY STAGE FUNDING
FOR JUNIOR INVESTIGATORS
BECAUSE, YOU KNOW, A LOT OF US
START WORKING ON SOMETHING AND
THEN WE SPEND THE LEST OF OUR
LIVES WORKING ON IT.
IF YOU CAN INVEST IN JUNIOR
FACULTY WHEN THEY ARE JUST
STARTING OUT AND REALLY TRYING
TO DIFFERENTIATE, TRYING TO FIND
WHERE THEIR CAREERS ARE GOING TO
GO THAT CAN BE EFFECTIVE BECAUSE
OFTEN YOU'LL HAVE SOMEBODY
DEVOTED TO YOUR ILLNESS FOR THE
REST OF THEIR CAREERS.
I THINK THAT'S A VERY GOOD WAY.
WE'VE ALSO FOUND IN MANY WAYS
THAT THE PATIENT FOUNDATIONS
OFTEN BRING US TOGETHER, JUST
LIKE THE NIH DOES IN CERTAIN
WAYS FOR DIFFERENT GROUPS.
FOR SMALLER GROUPS LIKE RARE
DISEASES PATIENT GROUPS BRING
PEOPLE TOGETHER AND THAT CAN
ENERGIZE EVERYONE AND GET PEOPLE
EXCITED AND SOMETIMES ONCE
OUTSIDERS SEE OR PEOPLE AREN'T
MAYBE IN THE NEUROLOGY COMMUNITY
START TO SEE WHAT PROTEINS ARE
BEING WORKED ON, WHAT PATHWAYS,
THAT BRINGS IN BASIC SCIENTISTS
AND THEY BECOME INTERESTED.
SO ENGAGING IT'S A -- ENGAGING
LOTS OF TYPES OF PEOPLE, REALLY
ENGAGING SCIENTISTS WHO MIGHT
HAVE A SHARED INTEREST.
AGAIN, IT'S BEEN BROUGHT UP
BEFORE, A LOT OF DISEASES
CROSS-FERTILIZE OTHERS, IF
YOU'RE WORKING ON ALS YOU'LL
LEARN SOMETHING ABOUT
PARKINSON'S DISEASE OR EXAMPLES
LIKE THAT.
BRINGING PEOPLE TOGETHER AND
SUPPORTING EARLY STAGE
INVESTIGATORS I GUESS WOULD BE
TWO OF THE MAIN WAYS THAT I
THINK ARE VERY HELPFUL.
>> THERE ARE OPPORTUNITIES WHEN
YOU'RE DEALING IN RARE DISEASES
TO TAKE ADVANTAGE OF THE FACT
THAT THE SAME TYPE OF DRUG MIGHT
BE POTENTIALLY TESTABLE IN
MULTIPLE DISEASES.
AND THAT CAN BE A REAL
OPPORTUNITY TO BRING PEOPLE
TOGETHER, EVEN WITHIN THE SAME
TRIAL AND SORT OF GET THE POWER
IN NUMBERS.
>> I CAN CHIME IN.
I THINK ONE OF THE THINGS THAT'S
BEEN VERY INTERESTING IS WHEN
SOME OF THESE PATIENT ADVOCACY
GROUPS REALLY EDUCATE A COMPANY
ABOUT A DISEASE BECAUSE MOST OF
THE TIME THE COMPANIES ARE NOT
NECESSARILY AWARE OF EVERYTHING
WE'VE HAD THAT WITH THE
FOUNDATION, INSTRUMENTAL IN
HELPING US, INFORMING US WHAT IS
THE NATURAL HISTORY, THE THINGS
WE NEED TO ADDRESS, THEY HELPED
US ON TRIAL DESIGN.
WHAT WOULD BE TOO BURDENSOME.
ANOTHER EXAMPLE WAS THE SIN GAB
ONE FOUNDATION.
WE LOOKED AT IT, FIVE YEARS AGO,
WE DIDN'T THINK THERE WERE
ENOUGH PATIENTS TO DO A CLINICAL
TRIAL.
THERE WAS THE SIN GAP RESEARCH
FOUNDATION, MIKE WAS RELENTLESS.
NOT ENOUGH PATIENTS
HERE'S THE PATIENTS WE FOUND.
OH.
HOW ABOUT IF WE DO THE NATURAL
HISTORY, SO YOU KNOW WHAT THE
NATURAL HISTORY IS.
AND THEY FUNDED AN ANIMAL MODEL
THAT WE'RE USING NOW TO TEST ONE
OF OUR CLINICAL CANDIDATES THAT
WE HOPE TO GET BY NEXT YEAR INTO
THE CLINIC.
THAT FOUNDATION WAS ABSOLUTELY
INSTRUMENTAL, FIRST GETTING
PEOPLE AWARE THIS DISEASE
EXISTS, AND THERE ARE A NUMBER
OF PATIENTS AFFECTED, BUT ALSO
UNDERSTANDING THE NATURAL
HISTORY.
AND GIVING SMALL GRANTS, YOU
KNOW, AS WE HEARD FROM JIM
BEFORE, ABOUT, YOU KNOW, FUNDING
EARLY STAGE RESEARCH.
THEY WERE THE DRIVING FORCE TO
REALLY ADVANCE THERAPIES.
>> NOW, I CAN JUST ADD ONE SMALL
SORT OF TECHNICAL ASPECT.
NIH, AMONG OTHER ORGANIZATIONS,
COLLECTED HUGE AMOUNTS OF
LIBRARIES OF DRUGS AND DRUG-LIKE
MOLECULES AND CAN BE SCREENED BY
HIGH-THROUGHPUT ASSAYS.
THE LIMITING FACTOR IS GENERALLY
THE ASSAY.
AND JUST EXPANDING ON WHAT WAS
SAID ABOUT YOUNG INVESTIGATORS,
FINDING SOMEONE WHO NOT JUST CAN
UNDERSTAND YOUR DISEASE BUT
FIGURE OUT A WAY OF MEASURING IT
IN A HIGH-THROUGHPUT WAY SO YOU
CAN SCREEN TENS OF THOUSANDS OF
COMPOUNDS TO SEE WHETHER BY
CHANCE ONE OF THEM MAY WORK IS
OFTEN A WAY TO GET A THERAPY
QUICKLY.
MANY OF THE ONES AT LEAST A FEW
OF THE LIBRARIES I KNOW HAVE
ALREADY UNDERGONE CLINICAL
TESTING AND THEY ARE KNOWN TO BE
SAFE.
IT DEPENDS A GREAT DEAL ON THE
SPECIFIC DISEASE, BUT THERE ARE
THESE NEW WAYS OF RAPIDLY
SCREENING COMPOUNDS.
AND FURTHERMORE, THERE ARE A
NUMBER OF CONTRACT RESEARCH
ORGANIZATIONS, MOST OF THE
CHEMISTRY NOW IS DONE IN FOREIGN
COUNTRIES.
AND YOU CONTRACT OUT THE
DEVELOPMENT OF SMALL MOLECULE
DRUGS TO BIND TO OR ACT ON A
SPECIFIC PROTEIN.
SO IF YOU HAVE AN ASSAY FOR A
PROTEIN, IT IS IMPOSSIBLE TO GET
A SMALL MOLECULE THAT YOU COULD
THEN TEST IN AN ANIMAL AND
ULTIMATELY A PERSON, IN A
RELATIVELY SHORT TIME.
AND I'M CURIOUS, DO YOU GUYS
HAVE COMMENTS ON THAT SORT OF
THING?
>> YEAH, WE HAD DONE ACTUALLY
SOME COLLABORATIVE WORK WITH THE
NIH, BECAUSE IT WAS LOOKING AT
ONE OF THE DRUGS WE DEVELOPED
FOR DRIVE A SYNDROME, COULD IT
WORK IN OTHER FORMS OF EPILEPSY
THEY HAVE A SYSTEM TO TEST THE
DRUG, COULD IT WORK IN OTHERS
FORMS.
WE JUST GAVE THEM THE DRUG AND
THEY COULD TEST, COULD THIS BE
USED IN OTHER INDICATIONS, SO
VERY VALUABLE.
>> I'LL ADD THE IMPORTANCE TO
EMPHASIZE IMPORTANCE OF NIH
FUNDING BASIC RESEARCH, THROUGH
DE NOVO DRUG DESIGN, YOU DON'T
HAVE TO SCREEN.
THINK ABOUT THE CHEMICAL SPACE,
ITS INFINITE ALMOST.
WHAT ARE THE ODDS WE ONLY
COVERED A FRACTION, BUT WITH ALL
THE COMPUTER STRUCTURAL
PREDICTIONS NOW IF YOU
UNDERSTAND A LOT OF BASIC
BIOLOGY, WHERE YOU WANT TO
TARGET, YOU CAN MAKE DE NOVO
DESIGN AND THERE'S EVEN WAYS TO
SCREEN WITH USING ARTIFICIAL
INTELLIGENCE METHODS.
SO THERE'S A LOT OF WAYS WE CAN
GET THROUGH SOME EARLY HURDLES,
EVEN FOR RARE DISEASES,
UNDERSTANDING BASIC BIOLOGY IS
CRITICAL TO FIND THE
DISEASE-MODIFYING THERAPIES THAT
ARE IMPORTANT.
>> WE'RE GETTING NEAR THE END.
I'D LIKE TO OPEN THIS UP TO
QUESTIONS FROM THE AUDIENCE.
I'M VERY INTERESTED IN WHAT YOU
GUYS WOULD LIKE TO KNOW ABOUT
THE SCIENCE AND TAKING THE
SCIENCE INTO COMMERCE.
>> [OFF MICROPHONE]
, IF I'M NOT MISTAKEN A THIRD OF
THE YOUNG PEOPLE HAVE LEFT
SCIENCE WITHIN FIVE YEARS.
SO, HOW DO WE MAKE SURE THAT
THAT INVESTMENT ACTUALLY PAYS
OFF?
>> I CAN START WITH THAT.
THE OTHERS WILL HAVE MORE.
>> HOW DO WE KEEP TRAINING AND
KEEP THEM IN SCIENCE?
>> AGAIN, THERE'S A LOT OF
REALLY TALENTED PEOPLE, THERE
REALLY ARE FUNDING CHALLENGES
EARLY ON.
AGAIN, FOUNDATIONS ARE CRITICAL,
ASPECTS OF EARLY FUNDING.
SO IS THE NIH IN TERMS OF EARLY
AWARDS LIKE K AWARDS.
INSTITUTIONS LIKE OURS HAVE TO
DO MORE SUPPORTING JUNIOR
FACULTY BECAUSE YOU'RE RIGHT,
WE'RE LOSING A LOT OF TALENTED
PEOPLE.
IT'S VERY EXPENSIVE TO GET
PEOPLE TO THAT POINT.
ONCE THEY DROP OFF IT'S HARD TO
COME BACK.
I THINK IT'S ABSOLUTELY CRITICAL
THAT NOT ONLY FOUNDATIONS, THE
NIH, BUT ALSO OUR OWN
INSTITUTIONS STEP UP AND MAKE
SURE WE SUPPORT OUR JUNIOR
FACULTY MORE BECAUSE WITHOUT
THEM REALLY EVERYTHING WILL COME
TO A HALT.
>> I THINK WHAT HAPPENS IS YOU
SEE THIS TRANSITION, PEOPLE WILL
GET A TRAINING GRANT.
YOU'VE GOT TO FIRST THE FIRST
R01 TO SECURE YOUR FUNDING TO
CONTINUE.
THERE'S SUCH AN EMPHASIS ON
CLINICAL CARE, SO MUCH PRESSURE
THAT THE UNIVERSITIES PUT ON
THESE JUNIOR FACULTY, YOU SHOULD
DO BOTH, YOU KNOW.
AND I'VE HAD THAT PRESSURE.
I HAD TO BE VERY FIRM, NO, I
NEED 70% OF MY TIME PROTECTED TO
STAY IN THE LAB SO I CAN GET MY
GRANT.
IF I'M DOING 50% OF CLINICAL
WORK THAT'S NOT ENOUGH TIME TO
GET MY GRANT.
YOU HAVE TO BE ABLE TO PROTECT
THESE PEOPLE TO ALLOW THEM TO DO
THE RESEARCH THAT THEIR CAREERS
CAN GROW.
>> I'LL MAKE A COUPLE COMMENTS.
IN StrokeNet WE HAVE A
TRAINING PROGRAM.
WE HAND PICK PEOPLE WHO ARE
REALLY EXCITED ABOUT RESEARCH.
WE'VE HAD GREAT TURNOUTS, 75%
STAYING INVOLVED AT LEAST
ENROLLING IN TRIALS, HALF
APPLYING FOR NIH GRANTS, GREAT
STATISTICS.
I KNOW IT'S A TOUGH TIME OF
UNCERTAINTY, OBVIOUSLY THIS IS A
SHAKY TIME BUT I HAVE TO BELIEVE
THAT'S GOING TO PASS.
REGARDSLESS, WE WANTED TO
UNDERSCORE THE POINT THAT
FOUNDATIONAL SUPPORTS, WORKING
DIRECTLY, FOR EXAMPLE THE
INSTITUTION I'M AT, AT YALE,
WORKING ON CREATING A SITUATION
WHERE WE SEE THE MOST PROMISING
TALENT AND INVEST IN THEM
DIRECTLY THROUGH PHILANTHROPIC
FUNDS.
IN YOU KNOW AN AREA, YOU WANT TO
ASSIST WITH INVESTING YOU COULD
WORK WITH INSTITUTIONS AS WELL
TO CREATE THAT PATHWAY.
SOMETIMES IT'S NOT A HUGE SCALE
OF MONEY BUT ENOUGH TO STAY ON
THE RESEARCH TRACK AND HAVE
STABILITY FOR THE NEXT FIVE
YEARS.
>> THE PROBLEM IS ACUTE IN
RESEARCH UNIVERSITIES THAT ARE
NOT MEDICALLY ATTACHED.
SUCH AS MY OWN AT M.I.T.
THE YOUNG FACULTY MAY BE
BRILLIANTLY EXPERIENCED IN HOT
NEW TECHNIQUES, NEW WAYS OF GENE
THERAPY, BUT THEY HAVE NO IDEA
OF THE DISEASES THAT COULD BE
TREATED.
WHAT WE DON'T DO A GOOD JOB OF
IS EDUCATING THE PhDs IN
HUMAN DISEASE IN GENERAL, SO
THAT WHEN A FOUNDATION OR GROUP
OF PATIENTS WANT TO FUND SOMEONE
WHO COULD WORK ON A DISEASE,
THERE'S NO WAY OF CONNECTING THE
PEOPLE.
AND I DON'T HAVE A SIMPLE
ANSWER.
BUT IT'S BEEN A PROBLEM FOR A
LONG, LONG TIME.
UNLESS YOU'RE TRAINED IN A
PARTICULAR AREA, YOU WOULDN'T
KNOW THE DISEASE.
>> A QUICK FOLLOW-UP TO THAT.
WE COULD WALK TO M.I.T. FROM
MASS GENERAL.
AGAIN, OUR INSTITUTIONS HAVE TO
DO A BETTER JOB.
I COULD WALK TO HARVEY'S LAB.
WE SHOULD DO IT MORE.
>> I THINK WE'VE LOST TRACK OF
WHAT I WAS GOING TO SAY.
GOING OFF TRACK.
BUT I'M JILL WOOD, MY COMPANY IS
PHOENIX NEXT, I CAME FROM THE
PATIENT ORGANIZATION ADVOCACY
WITH MY FOUNDATION, SO BEING
HERE FOR 15 YEARS NOW, I THINK A
LOT OF PEOPLE IN THIS ROOM ARE
NEW, AND DON'T KNOW WHAT
HIGH-THROUGHPUT SCREENING IS AND
DON'T KNOW WHAT LIBRARIES ARE,
DON'T KNOW WHAT ASSAY
DEVELOPMENT IS.
SO I THINK MAYBE TO EDUCATE SOME
OF THE NEW PEOPLE HERE MAYBE IT
WOULD BE GOOD TO EXPLAIN WHAT
HIGH-THROUGHPUT SCREENING IS.
>> I CAN DO IT.
OFTEN -- THERE ARE MANY WAYS OF
DOING IT BUT THE MOST COMMON IS
YOU HAVE A DISH, A CULTURE DISH,
WITH ROUGHLY 100 LITTLE TINY
CULTURES.
AND YOU ADD A DIFFERENT DRUG TO
EACH ONE.
EACH OF THE LITTLE TINY CULTURES
OF CELLS.
YOU CAN DO THIS ON A HUNDRED
DISHES, SAY YOU HAVE 10,000
DRUGS BEING SCREENED.
YOU HAVE TO HAVE A VERY QUICK
WAY OF RECOGNIZING WHETHER
THERE'S AN EFFECTIVE RESPONSE TO
THE CELL.
OFTEN THE CELLS CAN BE GENERALLY
GENETICALLY ENGINEERED SUCH THAT
IF A PARTICULAR PROTEIN IS
ACTIVATED, THE CELL TURNS RED.
SOMETHING LIKE THAT.
SIMILARLY, YOU CAN ASK IF I
INHIBITED A PARTICULAR GENE
WOULD THAT HELP MY DISEASE?
AND THE NEW CRISPR TECHNOLOGY
ALLOWS YOU TO DO THAT.
THAT IS, YOU GENETICALLY
ENGINEER THE CELL AND PUT THEM,
AGAIN, IN THESE CULTURE DISHES
WITH NEARLY 100 DIFFERENT
CULTURES, AND EACH GROUP OF
CELLS GETS A DIFFERENT CRISPR
RNA.
AND YOU KNOW WHAT GENE IS BEING
INLIBERTIED IN THAT CELL, YOU
CAN SEE WHETHER THERE'S AN
EFFECT.
SO, THERE ARE NEW TECHNOLOGIES
THAT ALLOW YOU TO SCREEN LOTS OF
THINGS VERY QUICKLY.
YOU CAN SCREEN GENES, YOU CAN
SCREEN SMALL MOLECULES.
AND I DON'T KNOW -- WELL, THIS
IS WHERE YOU NEED TO -- THE
LIBRARIES ARE AVAILABLE.
YOU CAN ORDER THEM.
MANY INSTITUTIONS HAVE THEM.
THE KEY IS DEVELOPING THE ASSAY
WHICH IS YOU NEED TO KNOW WHAT
THE GENE AND PROTEIN DO, WHETHER
YOU WANT TO JUST MEASURE THE
PROTEIN ITSELF OR YOU WANT TO
MEASURE THE PROTEIN IN A LIVING
CELL.
AND IF SO, IS IT A BRAIN CELL,
NERVE CELL, WHAT KIND OF CELL?
SO THERE'S A LOT OF SCIENCE THAT
HAS TO GO INTO DEVELOPING.
BUT, AGAIN, IF I GO TO THE
CYSTIC FIBROSIS MODEL, THE KEY
WAS DEVELOPMENT OF THE ASSAY
CELLS, WHERE IF THE MUTANT
CYSTIC FIBROSIS PROTEIN WAS
FUNCTIONAL, THE CELL WOULD
CHANGE COLOR.
AND THAT REQUIRED SOME VERY
CLEVER ENGINEERING WHICH WAS
DONE AT AURORA BIOSCIENCES WHICH
IS WHY VERTEX BOUGHT THE
COMPANY.
OKAY.
KEEP GOING.
>> WE'RE WORKING WITH NATIONAL
SCIENCE FOUNDATION ON
ESTABLISHING A REGIONAL
INNOVATION, ST. LOUIS, FOCUSED
ON NEUROTECH.
WITH OUR VENTURE DEVELOPMENT
ORGANIZATION WE SEE 50% OF OUR
BIOPHARMA PORTFOLIO FROM WashU
SCHOOL OF MEDICINE.
I WONDER IN TERMS OF BEST
PRACTICES, TALKING ABOUT YOUNG
INVESTIGATORS, HOW TO ALIGN
INCENTIVES INSIDE ACADEMIC
RESEARCH INSTITUTIONS FOR
COMMERCIALIZATION, WE'RE STILL
DEALING WITH THE CULTURE EFFECT
OF PUBLICATIONS, GRANTS, AND AS
A METRIC FOR SUCCESS VERSUS
COMMERCIALIZATION, UNDERSTANDING
WE'RE IN A NEW UNIVERSE NOW BUT
ANY THOUGHTS ON HOW TO ALIGN
CULTURE CHANGE THAT'S NEEDED TO
COMMERCIALIZATION?
>> YEAH, LET ME SPEAK TO THAT.
THIS IS WHAT I DO A LOT
LECTURING ON THIS THROUGHOUT THE
WORLD.
IT'S MOTIVATING AND EDUCATING
FACULTY.
WHEN I JOINED THE BOARD AT
CHILDREN'S HOSPITAL IN BOSTON
YEARS AGO, WITH TECHNOLOGY
LICENSING OFFICE WE ORGANIZED A
MONTHLY LUNCH TALK TO THE
FACULTY AND SENIOR POSTDOCS AND
STAFF ON THE ELEMENTS OF THE
BIOBUSINESS.
WHEN I MEET WITH GROUPS OF
FACULTY, I OFTEN ASK THEM HOW
MANY OF YOU CAN TELL ME ABOUT A
PATENT?
THE ANSWER IS ABOUT 5%.
SO, EDUCATING THE FACULTY, I
WOULD ARGUE, IS A VERY IMPORTANT
WAY OF DOING IT AND FOR THAT
MATTER LOOKING AT YOU, DOING IT
AT NIH, I THINK WOULD GO A LONG
WAY TO AT LEAST ENABLING THE
FACULTY TO REALIZE HOW THEY CAN
BEST IMPACT COMPANIES.
AND IT'S NOT ALWAYS BY TAKING
RESEARCH FROM THEIR LABS INTO
COMPANIES, BUT MUCH MORE OFTEN,
WHAT I DO, AND MY COLLEAGUES DO,
WE SERVE AS ADVISORS TO THE
COMPANIES.
WE HAVE SHARES IN THE COMPANY.
WE CAN TELL THEM OF NEW
DEVELOPMENT.
COMPANIES ARE FOCUS IT'S ON A
PARTICULAR AVENUE, AS ACADEMICS
WE CAN TELL THEM, HEY, HERE IS A
PAPER YOU SHOULD READ THEY WOULD
NEVER NOTICE.
ALSO THE FACULTY WILL HAVE
CONTACT AT THE YOUNG PEOPLE WHO
WORK IN THE COMPANIES NEVER
WOULD HAVE.
ACKNOWLEDGING START-UP, YOU CAN
GO A LONG WAY TO TAKING THESE
DEVELOPMENTS INTO COMMERCIAL
PRACTICE.
THE INVESTORS WANT TO HEAR THIS.
>> THE BIGGEST CHALLENGE BIOTECH
COMPANIES HAVE, TALKING WITH
ACADEMICIANS IS YOU REMEMBER THE
CURRENCY IN ACADEMIA IS
PUBLICATION.
AND MANY, MANY, MANY TIMES
THERE'S A GREAT APPROACH AND WE
REALIZE WE HAVE NO PATENT
PROTECTION, NO INTELLECTUAL
PROPERTY BECAUSE THEY PUBLISHED
ON IT, IT'S OUT THERE.
A COMPANY IS NOT GOING TO TAKE
THAT IF YOU CAN'T CONTROL THE
PATENT ON THAT.
THAT'S A BIG DEAL.
IF YOU HAVE SOMETHING YOU THINKS
IS COMMERCIAL, I LEARNED AT MASS
GENERAL, BEFORE WE COULD PUBLISH
ANYTHING WE HAD TO TAKE IT TO
THE PATENT LAWYERS, THEY WOULD
PATENT BEFORE WE COULD PUBLISH.
>> THE TIME IS RIGHT FOR MAKING
THESE APPROACHES, WE'RE LOOKING
TO DIVERSIFYING FUNDING SOURCES.
MAJOR UNIVERSITIES GET THAT.
MANY HAVE THESE VENTURE
ORGANIZATIONS, OFTEN THAT ARE
JOINT FACULTY AT OUR
INSTITUTIONS, AND I'LL SAY EVEN
NOW THINGS HAVE CHANGED WHERE WE
GET SORT OF ACADEMIC POINTS FOR
THINGS LIKE PATENTS, AND SO THE
FRAMEWORK IS CHANGING.
>> I THINK YOUR QUESTION IS
INTERWOVEN WITH THE QUESTION
ABOUT YOUNG FACULTY.
WHEN I SERVED ON THE TENURE
COMMITTEE YEARS AGO WE HAD
PUBLICATIONS, PEOPLE WERE
FINDING OUT THE TIME TO SPEND IN
DEVELOPING NEW TECHNOLOGY, NEW
DRUGS AND SO FORTH, DIDN'T COUNT
BECAUSE IT'S BEEN A LOT OF
YEARS, LESS PUBLICATIONS, BUT
MORE PATENTS.
WE HAD TO MEET WITH THE DEAN OF
THE COLLEGE OF MEDICINE, WELL,
WE NEED TO REVISE THIS, THE
CRITERIA.
AND SO FOR INSTITUTIONS THAT
DON'T HAVE IT, WHAT WE'RE GOING
TO BE SEEING IS A LOT OF JUNIOR
FACULTY LEAVING.
WHAT WE ENDED UP HAVING IS A
CONSIDERATION OF NEW DEVELOPMENT
PATENTS AS SOME CONDITIONS,
CRITERIA FOR PROMOTING STAFF,
RATHER THAN JUST PUBLICATIONS.
WE WERE ALSO CONSIDERING
PUBLICATIONS FOR THE NEW -- FOR
DISCLOSURES AS WELL.
SO IF INSTITUTIONS ARE ABLE TO
INCORPORATE THAT, INTO THE
PROMOTION AND TENURE PROCESS, IT
WILL KEEP MULTIPLE ENGAGED.
>> THANK YOU.
>> TWO MORE QUESTIONS, I'VE BEEN
TOLD WE'RE RUNNING OVER TIME.
>> THANK YOU.
CLAIRE RILEY, HERE IN PART AS
NEUROLOGIST, NEUROIMMUNOLOGIST,
DIRECTOR OF THE MULTIPLE
SCLEROSIS CENTER AT COLUMBIA.
ALSO SIT ON THE SCIENCE
COMMITTEE OF THE AMERICAN
ACADEMY OF NEUROLOGY.
I WANTED TO FOCUS ON DR. KHATRI,
ON YOUR COMMENTS ABOUT
LEVERAGING MULTI-ARMED TRIALS
AND IMPORTANCE OF THIS
INNOVATION, IN MULTIPLE
SCLEROSIS WE'VE HAD INNOVATION
IN THE LAST 30 YEARS, SEEING
MANY SUCCESSES.
I THINK ONE OF THE THINGS THAT
IS STIFLING THE NEXT STEPS FOR
EXAMPLE IN PROGRESSIVE M.S. IS
LACK OF ABILITY TO CORRAL MANY
INDUSTRY PLAYERS INTO WORKING
TOGETHER.
SO THAT WE CAN MINIMIZE AND THIS
REALLY SPEAKS TO OUR PATIENT
ADVOCACY GROUPS IN THE ROOM, AND
THANK YOU FOR THE PREMIUM WORK
YOU DO BUT TO MINIMIZE THE
IMPACT OF THE PLACEBO ARM OF THE
COMPARATOR ARM IN OUR CLINICAL
TRIALS REALLY SO THAT CARE CAN
BE ADVANCED FOR OUR PATIENTS AS
RAPIDLY AS POSSIBLE WITH AS FEW
PEOPLE EXPOSED TO PLACEBO, FOR
EXAMPLE, IN PROGRESSIVE M.S.
YOU YEARN FOR A WAY TO DO THIS
IN PHASE 3, PARTICULARLY AS I
SEE COMPANY AFTER COMPANY, YOU
KNOW, POURING MILLIONS OF
DOLLARS INTO THESE LARGE TRIALS
WITH YET ANOTHER PLACEBO ARM
WITH ALMOST THE EXACT SAME
INCLUSION AND EXCLUSION CRITERIA
BECAUSE TRIALS ARE SO SIMILAR.
HOW CAN WE MAKE THIS BETTER
TOGETHER AND AMONG OTHER THINGS
I THINK ABOUT OUR MECHANISMS FOR
IMPROVING CLINICAL TRIALS
DESIGN, FOR EXAMPLE PART OF OUR
CTSA AT COLUMBIA, I HOPE WE CAN
FIND WAYS TO DO THIS.
WOULD LOVE TO HEAR YOUR
THOUGHTS.
>> IT'S A REALLY IMPORTANT
QUESTION IN TERMS OF DOING THESE
OVER AND OVER, ONE AND DONE
TRIALS LIKE YOU SAID WITH
ONE-TO-ONE PLACEBOS, 50% NOT
GETTING AN ACTIVE THERAPY.
THERE ARE A TON OF OPPORTUNITIES
TO MAKE THINGS MORE EFFICIENT.
I'LL GIVE TWO EXAMPLES.
ONE IS OBVIOUSLY HAVING SOME
SORT OF CONSORTIUM, BRINGING
PEOPLE TOGETHER THE WAY YOU'RE
TRYING TO DO WITH THESE
COMPANIES.
IDEALLY WHAT YOU WOULD HAVE IS A
SITUATION WHERE THE COMPANY CAN
THEN BRING THEIR DRUG AND YOU
COULD HAVE MULTIPLE ARMS WITH
EACH DRUG AND ONE PLACEBO.
SOMETIMES THAT CAN SELL BECAUSE
IT'S RISK MITIGATION IN TERMS OF
INVESTMENT BUT SOMETIMES IT'S
NOT GOING TO WORK BECAUSE THEY
DON'T WANT TO COMPETE AND I'VE
BEEN IN THAT SCENARIO.
WHAT HELPED IN StrokeNet IS
POWER IN SCALE.
YOU WON'T GET TRIALS DONE
EFFICIENTLY IF YOU DON'T JOIN
OUR BANDWAGON.
THAT'S ONE POSITIVE ASPECT.
I WILL SAY THE SECOND THING AS
YOU'RE THINKING ABOUT TRIAL
EFFICIENCY IS MORE AND MORE
WE'RE DOING ADAPTIVE DESIGNS,
IT'S ALMOST HARD TO IMAGINE A
SITUATION WHERE YOU SHOULDN'T DO
AN ADAPTIVE DESIGN IN A PHASE 3
TRIAL.
IN OTHER WORDS LET'S SAY YOU
HAVE THIS NEW DRUG FOR
PROGRESSIVE M.S. AND SAY I'M
GOING TO RANDOMIZE EVERYBODY
WITH X, Y, AND Z.
IT MIGHT BE WORKING IN A SUBSET,
IF YOU CAN PRE-SPECIFY AND
ENRICH YOUR POPULATION AS YOU'RE
GOING ALONG YOU'LL INCREASE
CHANCES OF THAT TRIAL BEING
SUCCESSFUL.
THE KEY YOU HAVE TO PRE-SPECIFY
SO YOU DON'T KIND OF CHEAT IN
TERMS OF STATISTICAL PENALTIES.
OPPORTUNITIES LIKE THAT.
DEPENDING ON THE M.S. SOCIETIES,
WHETHER IT'S STRENGTH IN
BRINGING M.S. INVESTIGATORS
TOGETHER, WHETHER COMPASSIONATE
COLLEAGUES CHAMPIONING IN THE
COMPANIES TRYING TO GET PEOPLE
TO WORK TOGETHER.
I THINK THAT'S THE KEY TO
EVERYTHING WE DO.
I THINK ERIKA AUGUSTINE SAID
YESTERDAY ONE IMPORTANT THING
NINDS HAS DONE IS NOT JUST GIVE
US MONEY BUT BE CONVENERS TO
PULL US TOGETHER.
THAT RINGS TRUE TO ME.
>> MY NAME IS MARIA, A PATIENT
ADVOCATE IN THE LEUKODYSTROPHY
SPACE.
AMAZING PANEL.
I'M LUCKY, BASED IN
PHILADELPHIA, WORK A LOT WITH
THE GLIA RDRRN.
OUR YOUNG INVESTIGATORS ARE
SAVVY.
MY QUESTION AS WE TALK ABOUT K J
MULDOON, SHOUT OUT TO REBECCA, A
COLLABORATOR AND TREATED KJ,
WHAT I'M HEARING FROM YOUNG
INVESTIGATORS THEY WOULD LIKE A
MODEL MAYBE LED BY THE NIH WHERE
MEDICAL RESEARCH TEAMS ARE
REIMBURSED THROUGH PAYORS FOR
ONE AND DONE BUT DON'T HAVE
COMMERCIAL VIABILITY RIGHT NOW
BUT COULD ADVANCE THE SCIENCE ON
ONE AND DONE CASES AS INDUSTRY
SCALES UP AND MONETIZES, I WANT
TO ASK WHAT YOU THOUGHT OF THAT
IDEA.
>> EVERYONE IS LOOKING AT ME.
>> YES, I AM LOOKING AT YOU,
PROFESSOR LODISH.
YOU BROUGHT THIS UP.
>> I DON'T HAVE A CLEVER ANSWER.
>> I THINK AN INTERESTING
EXAMPLE YOU MENTIONED, HARVEY,
TRYING TO GO AFTER VERY RARE
INDICATIONS.
ONE OF THE THINGS THEY ARE DOING
IS THEY WILL GO AFTER A RARE
INDICATION ANDS DO A
PROOF-OF-CONCEPT IN A HUMAN TO
SHOW IT ACTUALLY WORKS.
SO WE'VE HAD THAT DISCUSSION
WITH HIM, OKAY, FOR A ROYALTY
PAYMENT THEN THE COMPANY CAN
TAKE THAT TO THE NEXT LEVEL AND
ACTUALLY DO IT FOR A LOT OF
DIFFERENT PEOPLE.
YOU'LL DO IT BECAUSE YOU'VE
SHOWN THAT IT WORKS IN HUMANS.
THAT'S THE BIG PROBLEM THAT ALL
COMPANIES HAVE, BIOTECH AND
PHARMACEUTICAL COMPANIES, HOW DO
REDUCE RISK?
HUNDREDS OF MILLIONS OF DOLLARS,
IT CAN FAIL.
AND, AGAIN, MOST OF THE PROGRAMS
THAT WE TAKE INTO THE CLINIC
WILL FAIL.
YOU HAVE TO LIVE WITH THAT.
IF YOU HAVE A PROGRAM YOU
ALREADY KNOW WORKS IN HUMANS,
THEN THE LIKELIHOOD OF SUCCESS
IS MUCH, MUCH, MUCH HIGHER.
THAT COULD BE A WAY OF SAYING
CAN WE, YOU KNOW, TREAT A COUPLE
PATIENTS, SHOW IT WORKS, MAYBE
USE THAT AS A FOUNDATION TO
EXPAND INTO MORE PATIENTS.
>> THANK YOU.
>> OKAY.
YOU GUYS ARE GREAT.
THAT'S ALL I CAN SAY.
THANK YOU VERY MUCH.
THANK YOU, WALTER, FOR GIVING US
THE PRIVILEGE BEING HERE AND
SPEAKING TO YOU.
AND I THINK IT'S TIME FOR LUNCH
>> THANK YOU, HARVEY.
THANK YOU, ED, CRAIG, POOJA, AND
EVARISTUS FOR A WONDERFUL
DISCUSSION.
I INVITE YOU TO PARTICIPATE IN
LUNCH BY THE AMERICAN BRAIN
COALITION IN 1175, RIGHT AROUND
THE CORNER.
CONTINUE THE GREAT DISCUSSIONS.
FIND THE SPEAKERS.
THEY ARE NOT GOING TO GET A
BREAK AT LUNCH.
SO MANY WONDERFUL THREADS
BROUGHT UP, I HOPE YOU'LL
CONTINUE.
FOR THOSE WHO ARE SIGNED UP FOR
ONE-ON-ONE DISCUSSIONS WITH SOME
OF OUR WONDERFUL PROGRAM STAFF,
YOU CAN COME TO DIANA OR TO NORA
AT REGISTRATION TO FIND OUT
WHICH ROOMS TO MEET AT FOR THE
BREAKOUT SESSIONS.
THANKS VERY MUCH.
>> GOOD AFTERNOON, FOLKS.
WELCOME BACK TO THE SECOND HALF
OF THE NON-PROFIT FORUM.
WE HAVE A GOOD PANEL COMING UP,
BUT A TOUGH ACT TO FOLLOW AFTER
WHAT WE SAW IN THE MORNING.
THEY ARE GOING TO BE UP TO IT, I
KNOW.
THIS PANEL WILL FOCUS ON
PUBLIC/PRIVATE PARTNERSHIPS AND
WE HAVE A NUMBER OF FOLKS WHO
HAVE LOTS OF EXPERIENCE IN
WORKING WITH DIFFERENT PARTS OF
OUR ECOSYSTEM HERE AT NINDS AND
NIH AT LARGE.
SO LET'S GET RIGHT INTO IT.
THE MODERATOR IS GOING TO BE
KRISTIN SCHNEEMAN, SENIOR
DIRECTOR AT FASTERCURES OF THE
MILKEN INSTITUTE.
OVER TO YOU.
>> I APPRECIATE THAT.
I'M A SENIOR DIRECTOR ON THE
FASTERCURES TEAM AT THE MILKEN
INSTITUTE.
SO EXCITED TO BE HERE.
SUCH A BIG FAN OF THE EVENT.
I HAVEN'T BEEN IN PERSON FOR A
NUMBER OF YEARS BUT TRY WATCH
VIRTUALLY AS MUCH AS I CAN EVERY
YEAR.
I WISH -- I'VE SECRETLY WISHED,
NOT NOT SO, THERE WERE MORE
EVENTS LIKE THIS, SO HONORED TO
BE ASKED TO PLAY A ROLE THIS
YEAR.
OUR TOPIC HERE IS OBVIOUSLY
AROUND -- AGENDA SAYS
PUBLIC/PRIVATE PARTNERSHIP.
WE'RE DEFINE THAT LOOSELY.
PUBLIC/PRIVATE PARTNERSHIP HAS A
TECHNICAL DEFINITION IN SOME
PARTS OF THE UNIVERSE BUT WE'RE
TALKING MORE LOOSELY ABOUT
PARTNERSHIPS THAT INVOLVE
PATIENT ORGANIZATIONS LIKE THE
ONES IN THIS ROOM AND PREVIOUSLY
PANEL SET US UP NICELY.
THANK YOU SO MUCH, DR. LODISH
AND PANEL, FOR HAVING TEED UP
CONVERSATION IN YOUR SESSION,
THE NEED FOR COLLABORATION AND
PARTICULARLY WITH PATIENT
COMMUNITIES.
I'LL JUST SAY A WORD ABOUT
FASTERCURES AND THE MILKEN
INSTITUTE.
IT'S A THINK TANK, NON-PROFIT,
NONPARTISAN, THAT COVERS A LOT
OF TERRITORY.
WE HAVE A HEALTH PILLAR, THAT
INCLUDES MY TEAM, FASTERCURES,
WHICH FOCUSES ON BIOMEDICAL
INNOVATION AND THE ECOSYSTEM,
HOW IT IS PERFORMING AND MAKING
IT GO FASTER TO SOLVE PROBLEMS
FOR EVERYBODY .
WE'VE HAD A LONG STANDING ROLE
WITH ORGANIZATIONS FROM OUR
BEGINNING, MORE THAN 20 YEARS
AGO.
AND I WON'T GO INTO REASONS FOR
THAT.
PEOPLE SOMETIMES WONDER WHY
BECAUSE PATIENT GROUPS HAVE LESS
RESOURCES, PERHAPS, THAN OTHER
PLAYERS ALWAYS, THAN OTHER
PLAYERS IN THE SYSTEM BUT WE'VE
ALWAYS THOUGHT THAT YOU ALL
BRING SOME VERY UNIQUE ASSETS
THAT OTHERS CANNOT REPLICATE IN
THIS SYSTEM, INCLUDING MONEY,
WHICH OBVIOUSLY OTHERS HAVE AS
WELL BUT PHILANTHROPY HAS UNIQUE
CHARACTERISTICS THAT CAN ALLOW
IT TO DO THINGS OTHER FORMS OF
CAPITAL CAN'T OR WON'T.
YOU HAVE ACCESS TO PATIENTS, TO
THEIR PARTICIPATION IN RESEARCH.
YOU CAN BE THE HONEST BROKERS
FOR THAT PARTICIPATION AND
RESEARCH, ALSO BRINGING INSIGHTS
INTO THE RESEARCH PROCESS.
AND THAT URGENCY WHICH WE'VE
HEARD ABOUT TODAY AND
REPRESENTATIVE WEXTON
EXEMPLIFIED VERY, VERY DIRECTLY
THE URGENCY, THE DRIVE, SOMEBODY
USED A TERM EARLIER, YOU'RE THE
TRUE DRIVERS OF PROGRESS.
THAT'S NOT JUST NICE TO HAVE.
IT'S A TANGIBLE ASSET GROUPS CAN
BRING TO THE PROCESS OF
DEVELOPING DRUGS.
WE'VE GOT AN AFFINITY NETWORK OF
PATIENT FOUNDATIONS WHERE WE
LIKE OTHERS IN THE ROOM TRY TO
BRING PEOPLE TOGETHER TO SHARE
WHAT THEY ARE DOING, BEST
PRACTICES, GOOD APPROACHES, NEW
BRICK WALLS YOU'RE RUNNING UP
AGAINST, HOW YOU'RE KNOCKING
THEM DOWN OR RUNNING AROUND THEM
OR VAULTING OVER THEM.
WE'VE DONE A LOT OF
COLLABORATION ACTUALLY FOUND A
PAPER WE WROTE, ALMOST 10 YEARS
AGO, OLDIE BUT A GOODY,
CORNERSTONES OF COLLABORATION,
FOUNDATIONS WERE SAYING WE GET
ASKED A LOT TO PARTICIPATE, NOW
WE'RE BEING ASKED TO CREATE THEM
AND DRIVE THEM MAYBE IN WAYS NOT
DONE BEFORE.
HOW ARE WE SUPPOSED TO THINK
ABOUT THAT?
THAT'S A LOT.
WE LOOKED AT ISSUES AROUND
COLLABORATION IN THAT REPORT.
NONE OF US CAN DO IT ALONE.
SCIENCE IS A TEAM SPORT,
STAKEHOLDERS ARE LOOK TO
FOUNDATIONS TO BE HONEST
BROKERS, TO LEAD COLLABORATIONS.
I HAVE AN AMAZING GROUP OF
SPEAKERS WITH A LOT OF
EXPERIENCE, LEADING AND ADVISING
AND CRAFTING AND SUPPORTING
PARTNERSHIPS OF MANY TYPES.
I'M GOING TO ASK EACH TO
INTRODUCE THEMSELVES AND SAY A
FEW OPENING WORDS IN A MINUTE.
WE'RE GOING TO TALK ABOUT A
NUMBER OF KEY QUESTIONS LIKE
WHEN IS COLLABORATION THE RIGHT
SOLUTION?
WHEN IS PARTNERSHIP THE RIGHT
SOLUTION?
WHAT FORMS MIGHT IT TAKE?
WE RECOGNIZE IT CAN BE HARDER
THAN DOING THINGS BY YOURSELF,
RIGHT?
HOW CAN FOUNDATIONS ENSURE THEIR
MISSION AND VALUES ARE PRESERVED
WHILE TAKING INTO ACCOUNT
PARTNERS' INTERESTS AND NEEDS?
WHAT ARE THE BIGGEST OBSTACLES
TO CREATING EFFICIENT AND
EFFECTIVE PARTNERSHIPS, EXAMPLES
OF WHEN IT'S WORKED WELL, WHAT
RESOURCES MIGHT BE NEEDED TO
ADDRESS KEY ISSUES OF THE THINGS
THAT KIND OF GREASE THE WHEELS
OF COLLABORATION, AGREEMENTS,
YOU KNOW, SOMETIMES IP, DATA
SHARING, ALL OF THOSE THINGS
THAT GO INTO WORKING WITH OTHER
STAKEHOLDERS IN THE SYSTEM.
WE'RE GOING TO LEAVE PLENTY OF
TIME FOR QUESTIONS.
IN THE QUESTIONS, I WANT TO
INVITE PEOPLE TO SHARE YOUR OWN
EXPERIENCES.
I KNOW SOME OF YOU IN THIS ROOM
I KNOW YOU RUN YOUR OWN SPACES.
IF YOU DON'T COME TO THE MIC I'M
GOING TO CALL ON YOU.
I'M GOING TO START, GO DOWN THE
ROW AND ASK THE SPEAKERS ON THE
PANEL TO INTRODUCE THEMSELVES
BRIEFLY, ONE SENTENCE ABOUT
YOURSELF, ONE ABOUT YOUR
ORGANIZATION.
WHO ARE YOU BRINGING INTO
PARTNERSHIP WITH WHAT GENERAL
AIMS, AND MAYBE AN EXAMPLE OF A
CURRENT EFFORT YOU'RE INVOLVED
WITH IF IT'S RELEVANT.
>> RACHEL WURSMAN,
NEUROSCIENTIST, NEUROETHICIST BY
TRAINING, BACKGROUND IN
NEUROMODULATION RESEARCH,
CURRENTLY WITH AMERICAN BRAIN
COALITION WORKING ON BUILDING
AND LAUNCHING EXTERNAL
STAKEHOLDER ENGAGEMENT
INITIATIVE.
AND ABC IS AN UMBRELLA
ORGANIZATION OF ADVOCACY GROUPS
THAT -- I'M LOOKING AT MARK,
HOPEFULLY I GET THIS
APPROPRIATE, PART OF THE MISSION
IS TO SPEAK WITH ONE VOICE TO
ADVOCATE ACROSS ORGANIZATIONS
AND ACROSS CONDITIONS AND ISSUES
WITHIN THE BRAIN HEALTH FIELD SO
THEY HAVE MEMBERSHIP.
AND MULTIPLE SECTORS AND
HUNDREDS OF ORGANIZATIONS.
SO, THE INITIATIVE THAT I'VE
BEEN WORKING ON IS TO HELP BUILD
PROGRAMS THAT CAN PROVIDE
SUPPORT TO, ON ONE HAND, BUILD
CAPACITY WITHIN ADVOCACY
ORGANIZATIONS FOR CONNECTING
PEOPLE WITH LIVED EXPERIENCE
WITH INDUSTRY, AND ALSO
PREPARING AND SUPPORTING
INDUSTRY TO BE ABLE TO PARTNER
WITH PEOPLE WITH LIVED
EXPERIENCE EARLY IN TECHNOLOGY
DEVELOPMENT, PRODUCT
DEVELOPMENT, SO THAT THE
PRIORITIES AND THE NEEDS AND THE
CREATIVITY FRANKLY OF THE PEOPLE
WHO ARE IN THIS ROOM AND WHO ARE
MEMBERS OF YOUR ORGANIZATIONS
CAN BE DRIVING INNOVATION.
AND SO THE -- SHOULD I WAIT TO
TALK ABOUT MORE OF THAT?
>> NO, PLEASE.
>> OKAY.
SO, THERE ARE A LOT OF EXCELLENT
PATIENT ENGAGEMENT, COMMUNITY
ENGAGEMENT RESOURCES OUT THERE.
MANY OF THEM PRODUCED BY FEDERAL
ORGANIZATIONS, BY MILLIKEN, AND
WE STILL RESPECTIVELY, THESE
ORGANIZATIONS ASSISTING WITH
THIS, HAVE PEOPLE AND
ORGANIZATIONS THAT SAY I WANT TO
DO COMMUNITY ENGAGEMENT, AND
WHERE DO I START?
AND AS WE KNOW, THE MAIN
CONSTRAINTS, AND WE DID A
MEMBERSHIP SURVEY, WE WERE NOT
SURPRISED BY RESULTS.
PART OF ONE OF THE THINGS WE
DISCOVERED IS REALLY THE MAIN
CONSTRAINTS BY ADVOCACY
ORGANIZATIONS AND START-UP MED
TECH COMPANIES AS WELL ARE
CONSTRAINTS IN TIME DUE TO
COMPETING PRIORITIES, AND
PERSONNEL LIMITATIONS, RIGHT?
SO I'M SURE A LOT OF THE
ORGANIZATIONS IN THIS ROOM
PROBABLY GET A LOT OF ENGAGEMENT
REQUESTS, AND EVEN JUST HAVING
THE RESOURCES OR INFRASTRUCTURE
TO EVEN RESPOND OR TRIAGE THOSE
REQUESTS, BUILDING CAPACITY
REALLY IS A COMPLEX THING.
AND SO WHAT ARE THE ELEMENTS?
PART OF WHAT WE DID WITH NEEDS
ASSESSMENT IS INTERVIEWING AND
WORKING WITH DIFFERENT ADVOCACY
ORGANIZATIONS, AND DISCOVERING
WHAT ARE THE TYPES OF SUPPORT
THAT WOULD BE ABLE THAT WE COULD
DELIVER IN A WAY THAT IS
EFFICIENT AND MINDFUL OF THOSE
CONSTRAINTS.
AND SO WE DISCOVERED A LOT OF
VERY INTERESTING THINGS, PART OF
THIS IS WHAT IS THE TRAINING,
RIGHT?
WHAT IS THE TRAINING, WHAT IS
THE ORIENTATION, WHAT IS NEEDED
BY ORGANIZATIONS, AND THERE'S A
REALLY WIDE VARIETY OF
CAPACITIES AND INVOLVEMENT IN
COMMUNITY ENGAGEMENT PROGRAMS,
AMONGST ADVOCACY ORGANIZATIONS.
AND FOR SOME, THERE WAS A DESIRE
TO KNOW MORE ABOUT WHAT IS THE
VALUE OF THIS AND HOW DOES THIS
RETURN VALUE TO OUR COMMUNITIES.
FOR OTHERS, THERE ARE QUESTIONS
OF HOW DO WE DO IT, RIGHT?
HOW DO WE ACTUALLY LOGISTICALLY
GET THIS STARTED.
AND SO WE HOSTED A COUPLE
GROUPS, AND OUT OF THAT PROCESS
WE'VE FOUND A NUMBER OF THINGS
THAT I THINK I'LL TALK ABOUT
LATER BECAUSE THEY BLEND WITH
SOME QUESTIONS THAT WILL BE
ASKED HERE.
BUT FOR BOTH THE INDUSTRY AND
ADVOCACY SIDE WE HAVE MEANINGFUL
INSIGHTS WE'VE GOTTEN FROM
PARTICIPATION FROM MANY OF YOU
IN THIS ROOM THAT WILL HELP US
TO PROVIDE FORMS OF SUPPORT, BE
IT THINGS LIKE LOGISTICS OR
CREATING FORUMS WHERE THERE CAN
BE SHARED LEARNING AMONGST
DIFFERENT ORGANIZATIONS WITH
DIFFERENT CAPACITY LEVELS.
A LOT OF THE ORGANIZATIONS THAT
HAVE COMMUNITY ENGAGEMENT HAVE
LEARNED A LOT OF LESSONS AND
DONE THINGS THE HARD WAY, AND
THEY HAVE REALLY REFINED THEIR
PROCESSES THROUGH TRIAL AND
ERROR OVER THE YEARS.
AND SO THERE'S A VALUE,
POTENTIALLY, IN SHARING SOME OF
THESE LESSONS AND SHARING SOME
TOOLS THAT HAVE BEEN DEVELOPED.
AND BY THE IDEA, THAT BY RAISING
CAPACITY REALLY KIND OF ACROSS
THE ECOSYSTEM, WE REALLY
RETURN -- VALUE CAN BE REALLY
RETURNED ACROSS THE ADVOCACY
ECOSYSTEM AS WELL AS ENGAGEMENT
AND TECHNOLOGY DEVELOPMENT EARLY
ON, RIGHT?
NOT JUST GIVING INPUT BUT, HEY,
WHAT'S THE PROBLEM YOU NEED US
TO SOLVE?
AND WHAT ARE THE QUESTIONS WE
SHOULD BE ASKING, AND WHAT ARE
THE THINGS WE HAVEN'T THOUGHT
OF?
AND HERE'S TECHNOLOGY WE'VE
DEVELOPED; HOW WOULD YOU USE IT.
RIGHT?
THESE QUESTIONS MAKE ALL THE
DIFFERENCE IN SOMETHING THAT
BECOMES ACTUALLY -- THAT'S
GREAT.
YOU FLAGGED A NUMBER OF THINGS
THAT WE'RE COMING TO.
ONE OF THE TYPES OF PARTNERSHIPS
WE MIGHT BE TALKING ABOUT HERE
IS AMONG PATIENT GROUPS, IN THE
FORM OF THE COALITION, HOW TO
LEVERAGE EACH OTHER'S STRENGTHS
AND TO SUPPORT EACH OTHER'S
CAPACITY.
YOU FLAGGED INDUSTRY
PARTNERSHIPS, OBVIOUSLY DR.
LODISH TOLD US ABOUT THE
FOUNDATIONAL CF FOUNDATION,
PEOPLE FIND IT HARD TO RELATE
BECAUSE THEY ARE SO BIG BUT THEY
ARE A RARE DISEASE GROUP AND THE
VALUE THEY PROVIDED TO AURORA
AND VERTEX WAS NOT JUST MONEY
BUT ACCESS TO THEIR CENTER'S
EXCELLENCE.
THEY KNEW THE PATIENTS, GOT THEM
INVOLVED IN TRIALS, HAD PATIENT
DATA IN THE REGISTRY, ALL THAT
HAD VALUE FOR THE COMPANY IN
ADDITION TO THE MONEY WHICH WAS
PROBABLY A DROP IN THE BUCKET OF
WHAT IT TOOK.
WE'LL COME BACK TO THOSE THINGS.
TO THE QUESTION HOW YOU GET
STARTED I'LL TURN TO JOE
VANDIGO.
TELL PEOPLE WHAT APPLIED P EX
DOES.
>> I'M A HEALTH RESEARCHER BY
TRAINING, MY CAREER HAS BEEN
ASKING PEOPLE DID YOU ENGAGE
PATIENTS, IF THEY SAY NO WE GET
TO WORK TOGETHER.
AT APPLIED PATIENT EXPERIENCE OR
JOB IS TO COLLECT PATIENT
EXPERIENCE DATA AND HELP FOLKS
APPLY TO MEET NEEDS, WHETHER
ADVOCACY MISSION, PRODUCT
DEVELOPMENT MISSION, WHATEVER
THE PURPOSE IS.
THE DOSSIER, THE KEY REASON I'M
HERE TO SPEAK TO YOU TODAY, BUT
THE SECOND PIECE WE ALSO WORK
WITH PATIENT GROUPS TO
OPERATIONALIZE PATIENT-LED
RESEARCH AS WELL.
SOMETHING THAT'S GROWN OUT OF
THAT THROUGH A COLLABORATION
WITH NATIONAL HEALTH COUNCIL,
PSMD, IS THIS CONCEPT OF A
PATIENT EXPERIENCE DOSSIER.
AND IT GREW OUT OF THIS DESIRE
FOR PATIENT ORGANIZATIONS TO
HAVE -- AGAIN TO YOUR POINT ON
JUST CAPACITY -- TO RESPOND TO
REQUEST, WHEN INFORMATION IS IN
20 PLACES THERE'S SOME NOTES
FROM A PATIENT, MEETINGS,
THERE'S SOME ON THE WEBSITE, OH,
TWO PAPERS PUBLISHED LAST YEAR,
AND THE GOAL OF DOSSIERS IS TO
TAKE THIS INFORMATION, COLLECT
IT INTO A FORMAT THAT'S
TEMPLATED AND CONSISTENT, AND,
AGAIN, CAN BE DONE IN A
MULTITUDE OF WAYS.
I'M HAPPY TO CONTINUE THE
DISCUSSION IN TERMS OF FIGURING
OUT BECAUSE WE'VE JUST BEGUN TO
PILOT IT, INTERESTED IN THOUGHTS
HOW TO OPERATIONALIZE.
THE END GOAL IS THIS IS
SOMETHING THAT CAN BEGIN THE
CONVERSATION BY TAKING THE DATA
THAT'S CURRENTLY KNOWN ABOUT THE
PATIENT JOURNEY INCLUDING
STARTING AT THE TIMES OF
DIAGNOSIS.
REPRESENTATIVE WEXTON SET THIS
UP AS SHE DESCRIBED HER JOURNEY,
HOW POWERFUL IT IS AND THE
FOUNDATIONAL PIECE TO UNDERSTAND
WHAT IS WHAT UNDERPINS
PARTNERSHIP AND RELATIONSHIP.
THE DOSSIER INCLUDES EXPERIENCES
WITH CURRENT TREATMENT,
PROVIDERS YOU'RE SEEING, HOW YOU
FEEL YOUR NEEDS ARE MET WITH
CURRENT SLATE OF TREATMENTS,
WHAT OUTCOMES ARE IMPORTANT TO
YOU.
GETTING THAT DOCUMENTED HELPS
GET WHAT'S KNOWN OUT THERE.
THAT'S THE PUBLICLY AVAILABLE
LIVING RESOURCE.
AND THE SECOND PIECE WE FOUND IS
THAT HELPFUL AS WE'VE PILOTED IT
I WAS GAPS.
WE'VE GOT A TON OF INFORMATION
WHAT HELPS FOLKS BE ADHERENT, A
TON OF INFORMATION ON DIAGNOSIS
JOURNEY BUT PERHAPS WE'RE
LACKING IN PERHAPS UNMET NEEDS,
OR HOW SOMEONE'S CONDITION MAY
AFFECT THEIR PERSONAL FINANCES,
ABILITY TO WORK, RELATIONSHIPS
WITH THEIR FAMILY.
COULD PARTNER TO FILL IN TERMS
OF NEED FOR FUTURE THERAPY.
>> THAT'S GREAT.
I LOVE THIS IDEA OF A TOOL THAT
CAN BE USED ACROSS A LOT OF
CONTEXTS.
IT MIGHT HAVE DIFFERENT MEANINGS
FOR DIFFERENT AUDIENCES, WE NEED
TO DO MORE IN AN AREA FOR
CERTAIN TYPE OF PARTNERSHIP BUT
I FEEL LIKE IT'S A GREAT WAY TO
GET STARTED TO THINK WHAT DO YOU
HAVE OF VALUE TO A NUMBER OF
STAKEHOLDERS.
THE OTHER THING WE CAN COME BACK
TO LATER IS ACTUALLY THE IDEA OF
VALUE.
A LOT OF CONVERSATION HERE IS
ABOUT THERAPEUTIC DEVELOPMENT
BUT A LOT OF GROUPS HAVE
APPROVED THERAPIES, IT HAS TO
GET PAID FOR AND PATIENT
ORGANIZATION JOURNEY IS NOT OVER
WHEN SOMETHING GETS THROUGH FDA
AND WE ALSO HAVE TO ASCRIBE
VALUE TO PAYERS AS WELL.
I'M GOING TO COURTNEY
SILVERTHORN FROM FOUNDATION FOR
NIH, MOST PEOPLE KNOW WHAT FNIH
IS, MAYBE NOT OUTSIDE THE ROOM
BUT ITS MIDDLE NAME IS
PUBLIC/PRIVATE PARTNERSHIPS.
THIS WAS A SPECIALTY FOR YOU
ALL.
I DO IT EXTRAORDINARILY WELL AND
ACROSS SPACES.
>> THANK YOU FOR THE COMPLIMENT.
COURTNEY SILVERTHORN, STRATEGIC
ALLIANCES AND INNOVATION.
FOUNDATION FOR NIH IS NOT THE
NIH.
WE ARE A SEPARATE 501(c)3
NON-PROFIT ESTABLISHED BY
CONGRESS.
WE'VE BEEN AROUND FOR ABOUT 27
YEARS.
AND OUR MISSION IS TO SUPPORT
THE MISSION OF THE NIH.
SO ANYTHING THAT TOUCHES HUMAN
HEALTH IS WITHIN THE SCOPE OF
OUR MISSION.
THAT IS SOMETIMES A CHALLENGE
AND OPPORTUNITY BECAUSE WHEN YOU
ARE -- THE AMERICAN HEART
SITUATION IS CLEAR WHAT YOU DO
WHEN YOU'RE THE FOUNDATION FOR
THE NIH, MAYBE NOT SO MUCH, BUT
WE DO HAVE A WIDE RANGE OF
PARTNERSHIPS ACROSS A NUMBER OF
DIFFERENT HEALTH SPACES.
REALLY OUR MISSION BOILS DOWN TO
THREE MAIN PILLARS.
ONE IS SUPPORTING SCIENCE.
WE DO THAT PRIMARILY THROUGH
PUBLIC/PRIVATE PARTNERSHIPS IN
THE PRE-COMPETITIVE SPACE.
THERE WAS A COMMENT IN THE
EARLIER PANEL ABOUT THE
CHALLENGES OF BRINGING MULTIPLE
COMPANIES TOGETHER, WHAT WE
FOUND IS THAT WHEN WE ARE
IDENTIFYING OPPORTUNITIES THAT
BENEFIT MORE THAN ONE COMPANY,
MORE THAN ONE SECTOR OF THE
FIELD, WE CAN BRING TOGETHER
ORGANIZATIONS THAT WOULD BE
COMPETITORS OUTSIDE OF OUR SPACE
TO COME AND WORK TOGETHER WITH
PATIENT GROUPS WITH THE NIH,
WITH THE FDA, TO REALLY MOVE THE
ENTIRE FIELD FORWARD IN A WAY
THAT BENEFITS EVERYONE.
WE ALSO HAVE PILLARS ON
SUPPORTING SCIENTISTS, AS WELL
AS HELPING DEVELOP TRUST IN
SCIENCE.
BUT IN THE CONTEXT OF PATIENT
ENGAGEMENT, WE HAVE REALLY
DOUBLED DOWN ON A COMMITMENT TO
100% PATIENT ENGAGEMENT IN EVERY
PUBLIC/PRIVATE PARTNERSHIP THAT
WE DEVELOP AND EXECUTE.
AND SO WHAT THAT MEANS FOR US IS
THAT EVERY PARTNERSHIP THAT WE
TAKE ON GOES THROUGH DESIGN
PHASE WHEN WE'RE BRINGING
STAKEHOLDERS ACROSS THE SPECTRUM
FROM BIOTECH LEADERS, PATIENT
GROUPS, PATIENTS WITH LIVED
EXPERIENCE, ACADEMICS, SO FORTH,
TO COME TOGETHER TO DEFINE THE
PROBLEM, WHAT IS THE WHITE SPACE
FNIH CAN MOVE SOMETHING FORWARD
IN, WHAT TYPES OF RESEARCH
REQUIREMENTS ARE NEEDED,
RESOURCES IN-KIND AND FINANCIAL,
WHAT ARE THE TIMELINES AND
DELIVERABLES BECAUSE MOST OF
WHAT WE DO IS FUNDRAISING AND
THEN PROJECT MANAGEMENT.
IN THE EXECUTION OF OUR
PROJECTS, WE REALLY STARTED TO
SET THE STANDARD IN ACCELERATING
MEDICINES PARTNERSHIP PROGRAM, A
SERIES OF PARTNERSHIPS, SOME
DATING BACK TO 2014, THE PROGRAM
CELEBRATED ITS 10-YEAR
ANNIVERSARY LAST YEAR.
THERE ARE LARGE SCALE
LONGER--TERM RUNNING BETWEEN 5
AND 7 YEARS, TO DEVELOP
RESOURCES, DATASETS, PROTOCOLS,
VALIDATED PROCEDURES, CONSENSUS
PROCESSES, IN A NUMBER OF
THERAPEUTIC AREAS.
EACH PARTNERSHIP IS A LITTLE
DIFFERENT BUT WITHIN THE AMP WE
HAVE PATIENT ADVOCATES, PATIENTS
WITH LIVED EXPERIENCE WHO SERVE
AS CO-CHAIRS OF THE STEERING
COMMITTEES.
FOR EXAMPLE, CARLOS IS A PATIENT
WITH LIVED EXPERIENCE, ALSO PART
OF NOMI, THE CO-CHAIR IN
SCHIZOPHRENIA, JANET CHURCH
PRESIDENT OF THE SHÖGREN'S
FOUNDATION WITH LIVED EXPERIENCE
CO-CHAIRS IN IMMUNE DISEASES.
PROTEIN THERAPY CONSORTIUM, WE
HAVE A NUMBER OF PATIENT
ADVOCACY GROUPS THAT SERVE ON
THE STEERING COMMITTEE AS WELL
AS A GROUP IN THE PORTFOLIO.
SOMETIMES BRINGING IN PATIENT
GROUPS FOR SPECIFIC POINTS
WITHIN THE PROJECT DURING THE
EXECUTION, TO SERVE IN A WORKING
GROUP, PROVIDE INPUTS.
SO REALLY SPANNING THE PANOPLY
OF THE WORK WE DO.
WE'RE BUILDING IN PATIENT
ENGAGEMENT, PATIENT INPUT AT
EVERY LEVEL, AND WITHIN THE LAST
3 YEARS AGO WE ALSO HAVE NOW A
DIRECTOR OF PATIENT ENGAGEMENT
WHO IS SERVING AS A CONSULTATION
OF SORTS, SIMILAR TO THE WORK
YOU'RE DOING BUT INTERNAL TO THE
FOUNDATION WHO IS HELPING THE
DESIGN TEAM, HELPING THE STAFF,
HELPING THE PROJECT STEERING
COMMITTEE, BE AS EFFICIENT AND
SUCCESSFUL AS POSSIBLE IN
GETTING THAT PATIENT INPUT
INCORPORATED INTO THE PROJECT.
>> THAT'S GREAT.
GOOD TO KNOW, EVERYONE SHOULD
KNOW THE PLATFORMS EXIST WHERE
YOU CAN COME INTO PARTNERSHIP
WITH A LOT OF OTHER KEY
STAKEHOLDERS IN THE COMMUNITY
AND INDUSTRY IS OFTEN PICKING UP
THE TAB.
SO YOU MAY NOT EVEN NEED TO
PARTICIPATE AT THAT LEVEL.
BUT, YEAH, VERY EFFECTIVE
PARTNERSHIPS WITH THE FNIH.
YOU DON'T HAVE TO ANSWER NOW
COURTNEY, BUT WE'D LOVE TO KNOW
IF YOU HEARD ANYTHING FROM
PATIENT ORGANIZATIONS OR PATIENT
PEOPLE WITH LIVED EXPERIENCES IN
THE COURSE OF ENGAGEMENTS THAT
WERE STRIKING OR CHANGED WHAT
THE AIMS OF THE PROJECT MIGHT BE
OR SOMETHING, YOU DON'T HAVE TO
ANSWER THAT NOW.
I'LL TURN TO SHEENA POSEY
NORRIS, NATIONAL ACADEMIES OF
SCIENCE, ENGINEERING, MEDICINE.
I THINK EVERYONE IS FAMILIAR
WITH THE INCREDIBLE WORK THE
NATIONAL ACADEMIES HAS DONE OVER
MANY YEARS AND RIGOROUS SCIENCE
THEY ALWAYS BRING TO THE FORE.
BUT THEY HAVE ALSO BEEN VERY
INTERESTED IN ACTION
COLLABORATIVES IN RECENT YEARS,
SUPER INTERESTING, AND SHEENA
TOLD US ABOUT ONE THAT'S
RELEVANT TO OUR TOPIC TODAY.
INTRODUCE YOURSELF AND TALK
ABOUT THAT.
>> THANK YOU SO MUCH.
THANK YOU, AGAIN, TO DR.
KOROSHETZ, TO KATIE, SHEILA, FOR
INVITING ME, TRULY HONORED AND
APPRECIATE THE SUPPORT FROM ALL
OF OUR SPONSORS REPRESENTED IN
THE ROOM AND MANY WHO
VOLUNTEERED.
I'M SHEENA POSEY NORRIS, I'M
ALSO SENIOR PROGRAM OFFICER IN
THE HEALTH AND MEDICINE ARM OF
THE NATIONAL ACADEMIES.
FOR CONTEXT IF YOU DON'T HAVE A
MASSIVE NATIONAL ACADEMIES BOOK
IN YOUR BOOK CASE, WHO WE ARE,
NATIONAL ACADEMIES PRIVATE
NON-PROFIT INSTITUTIONS PROVIDE
INDEPENDENT OBJECTIVE ANALYSIS
ON TOPICS IN SCIENCE,
ENGINEERING, MEDICINE, AN OLD
INSTITUTION, ESTABLISHED AND
CHARTERED BY PRESIDENT LINCOLN
IN 1863.
SO WE HAVE BEEN DOING THIS TYPE
OF WORK, KNOWN FOR CONSENSUS
STUDIES, PUTTING FOR
RECOMMENDATIONS TO THE COUNTRY
AND CONGRESS WHEN MANDATED.
WE HAVE HONORIFIC ARM OF THE
NATIONAL ACADEMIES, SEVERAL OF
YOU ARE PART OF IT, BECAUSE OF
YOUR CONTRIBUTIONS TO THE FIELD.
THAT IS THE NATIONAL ACADEMIES
ITSELF.
THE FORUM ON NERVOUS SYSTEM
DISORDERS WAS ESTABLISHED IN
2006, TO BRING TOGETHER THESE
DIFFERENT STAKEHOLDERS AND
LEADERS ACROSS CROSS SECTOR IN
NEUROSCIENCE, THE FEDERAL
GOVERNMENT, NINDS, SEVEN OTHER
I.C. DIRECTORS AND THE BRAIN
INITIATIVE.
I SAW JOHN NGAI EARLIER.
WE BRING TOGETHER NON-PROFIT
ORGANIZATIONS, THIS COULD BE
DISEASE ADVOCACY ORGANIZATIONS,
NON-PROFITS WITHIN THE SPACE OF
THE SIMON FOUNDATION, DANA
FOUNDATION, AS WELL AS
PROFESSIONAL SOCIETIES.
IN ADDITION TO INDUSTRY, PHARMA,
BIOTECH ARE REPRESENTED ON OUR
FORUM AS WELL AS ACADEMIA, WE'RE
CHAIRED BY DR. FRANCIS JENSON,
CHAIR OF NEUROLOGY, UNIVERSITY
OF PENNSYLVANIA, DR. DEANNA
BARCH, UNIVERSITY OF WASHINGTON.
THE FORUM IS MEANT TO PROVIDE
NEUTRAL VENUE.
WE'RE ALL CONNECTED.
THAT'S SO TRUE, ALL DEALING WITH
THE SAME ISSUES, DIFFERENT
REALMS AND TOPICS.
OUR JOB IS CREATE THAT SPACE FOR
EVERYONE TO COME TOGETHER, WHAT
ARE THE PRESSING ISSUES WE NEED
TO ELEVATE, THE TOPICS, SET THE
STAGE FOR FUTURE POLICY ACTION
WITHIN NEUROSCIENCE.
WE ARE DISEASE AGNOSTIC.
THERE ARE TIMES WE DO FOCUS ON
TOPICS SPECIFICALLY IN DISEASE
SPACES BUT A LOT OF OUR WORK
LOOKS AT THE BROADER LEVEL FROM
BASIC TRANSLATIONAL AND CLINICAL
SCIENCE TO WORKFORCE
DEVELOPMENT.
I'M HERE TO TALK ABOUT AN ACTION
COLLABORATIVE WE LAUNCHED TWO
YEARS AGO.
I HAVE TO GIVE CREDIT TO A
MEMBER OF OUR TEAM IN THE BACK,
DR. CHILDERS, AND KIMBERLY OGUN
IS IN D.C. NOW.
I HAVE TO RECOGNIZE THEM.
I'M THE FACE SITTING HERE; THEY
ARE CARRYING OUT THE WORK.
THESE ARE PARTICIPANT-DRIVEN
ACTION ORIENTED EFFORTS.
WE CONVENE AN ACTIVITY, A
WORKSHOP ON A TOPIC.
WE MAY PUT TOGETHER A GROUP OF
MEMBERS FROM PLANNING COMMITTEE
AS WELL AS EXTERNAL STAKEHOLDERS
THAT WANT TO PUSH FOR ACTIONS IN
THE SPACE.
FOR EXAMPLE, WE RECENTLY HAD A
WORKSHOP WITH THE MULTI-MODAL
BIOMARKERS, AND HAVE DEVELOPING
A PAPER RELEASED THIS YEAR
AROUND THAT TOPIC.
OUR ACTION COLLABORATIVE WAS
LIVED EXPERIENCE FOR STRONGER
NEUROSCIENCE, AND THE IMPETUS
FOR THIS ACTION COLLABORATIVE,
MERGED AS A RESULT OF THE
FORUM'S MEMBERS, WE HAVE 50
MEMBERS, ACROSS VARIOUS ACTORS
AND STAFF DESIRE TO ELEVATE THE
VOICE OF INDIVIDUALS WITH LIVED
EXPERIENCE IN THE WORK WE'RE
DOING.
WE'RE ALL TALKING ABOUT
DECISIONS, POLICY MAKING, BUT
MISSING THE PEOPLE WHO SHOULD BE
AT THE TABLE DRIVING THE
DECISIONS, SO THAT WAS THE
IMPETUS.
DR. NICOLE BOSCHY FROM THE
NATIONAL M.S. SOCIETY WAS
INSTRUMENTAL IN DRIVING THIS,
AND DR. TIM COOKSY TO PUSH THE
MECHANISMS TO PUT TOGETHER THIS
ACTION COLLABORATIVE.
WE SURVEYED INDIVIDUALS THAT WE
ENGAGED FOR WORKSHOPS, FEEDBACK
WAS WE WANT TO DO MORE.
OUR STAFF WORKED TO CREATE THE
MECHANISM.
I'LL ACKNOWLEDGE WE HAVE VERY
LARGE PATIENT ADVOCACY
ORGANIZATIONS RECOMMENDED BUT
THEY ARE NOT THE VOICE OF THE
INDIVIDUAL WITH LIVED
EXPERIENCE.
THAT WAS REALLY IMPORTANT TO US.
OBJECTIVE IS TO BRING TOGETHER
INDIVIDUALS WITH FIRST HAND
EXPERIENCE WITH MENTAL,
NEUROLOGICAL, VISUAL CONDITIONS
TO SHARE EXPERIENCE AND HELP
GUIDE THE WORK OF THE
NEUROSCIENCE FORUM, NOT JUST
OUTPUT, AFTER A MEETING HELPING
DISSEMINATE BUT BE PART OF THE
STRATEGY, WHAT TOPICS, ARE WE
GOING IN THE RIGHT SPACE, IS THE
SCOPE CORRECT, WHAT THINGS ARE
WE MISSING?
THE GOAL, OUR MEMBERS, WE HAVE
11 MEMBERS IN THE FIRST COHORT,
SERVES A 3-YEAR TERM, IN THE
FIRST COHORT, WELCOME TO NOT
SERVE BUT THEY ARE ENERGIZED AND
HAVE BEEN ENGAGED.
THEY PROVIDE FEEDBACK ON THE
SCOPE OF THE WORK, NOMINATE AND
SERVE ON PLANNING COMMITTEES AT
THE NATIONAL COMMITTEES FOR
OTHER ACTION COLLABORATIVES,
SOME MEMBERS SERVE ON CONSENSUS
STUDY COMMITTEES AS WELL TO HELP
WITH THE RECOMMENDATION
GENERATION WE DO AT THE
ACADEMIES.
THEY VOLUNTEER TO STATE AND
OTHER ACTIVITIES AS WELL AS
SHARE EXPERTISE AND PERSPECTIVE
ON EMERGING TOPICS.
THROUGHOUT THIS TIME, IT TOOK A
YEAR FOR THE PROJECT TO COME
INTO FRUITION.
WE WERE HAVING CONVERSATIONS
WITH ORGANIZATIONS THAT ALREADY
DO THIS.
THEY DO THIS WELL.
WHAT ARE THE THINGS WE NEED TO
DO?
WE'RE BABIES AT THIS.
HELP US, GUIDE US.
WE WON'T WANT TO WAIT LONG TO
GET EVERYTHING PERFECT.
WE WANT TO START AND DO IT
RIGHT.
SO WE HAD LENGTHY CONVERSATIONS
ACROSS MANY ORGANIZATIONS,
PATIENT COUNCILS, INDIVIDUALS,
TO HELP DRIVE HOW WE WERE GOING
TO HAVE THIS FUNCTION.
WE WORKED INTERNALLY TO SET UP
CASH PAYMENTS FOR INDIVIDUALS
PARTICIPATING, NOT BENEFITING
PROFESSIONAL FROM BEING PART OF
THIS OR ECONOMIC NEED FOR
ATTENDING MEETINGS, SO THAT WAS
A REALLY BIG PIECE THAT WE WERE
COMMITTED TO MAKING SURE WE
PASSED THAT.
I KNOW WE'LL GET LATER TO THE
VALUE AND IMPACT OF SOME WORK
BECAUSE I HAVE A LOT TO SHARE
BUT I WANTED TO SHARE THE NEW
COLLABORATIVE.
>> THANK YOU.
DISCIPLINE OF PATIENT ENGAGEMENT
REACHED THE HALLOWED HALLS OF
THE NATIONAL ACADEMIES.
WE'RE IN GOOD SHAPE, GOOD
STANDING HERE.
AND I THINK WE'VE HEARD THERE'S
A LOT OF OPPORTUNITY FOR PATIENT
COMMUNITIES TO ENGAGE IN
PARTNERSHIP WITH OTHER
STAKEHOLDERS THROUGH A LOT OF
MECHANISMS, ACTION COLLABORATIVE
AT NATIONAL ACADEMIES, AN AMP
PROJECT AT FNIH.
THROUGH, THROUGH, THROUGH.
HOW CAN THEY THINK ABOUT
PRIORITIZING?
THESE ARE THE ORGANIZATIONS WITH
THE LEAST AMOUNT OF RESOURCES
TYPICALLY TIME, YOU KNOW,
FOCUSED, HOW SHOULD GROUPS THINK
ABOUT HOW TO PRIORITIZE?
RACHEL, I WAS GOING TO START
WITH YOU.
>> I
>> ANOTHER THING I DIDN'T SAY
ABOUT OUR PROJECT, IT GREW OUT
OF A COLLABORATION BETWEEN ABC
AND THE FDA TAP PROGRAM.
AND WAYS WE CAN PROVIDE SUPPORTS
TO COMPANIES THAT HAVE
BREAKTHROUGH DEVICE DESIGNATION.
ALSO I WANT TO ACKNOWLEDGE KATIE
SALE AND JEN FRENCH, THE TWO
PEOPLE WITHIN THE ORGANIZATION
WHO HAVE REALLY DONE A LOT TO
SHEPHERD THIS AND WILL BE
CARRYING THE PROJECT FORWARD
EVEN AFTER THE END OF THE
FUNDING.
THE QUESTION, HOW ARE THE WAYS
THAT YOU CAN PRIORITIZE
REQUESTS?
SO I THINK WHEN IT COMES TO
TRIAGING REQUESTS, THIS IS
SOMETHING THAT WAS EXPRESSED AS
AN UNMET NEED GIVEN CONSTRAINTS,
HOW DO WE EVEN TRIAGE THESE
REQUESTS?
SOME ANSWERS THAT WE HEARD THAT
WERE SHARED BETWEEN
ORGANIZATIONS IS VETTING THEM
FOR THINGS LIKE THERE'S SOME
ORGANIZATIONS THAT WON'T
ENTERTAIN ENGAGEMENT REQUESTS IS
THE PROJECT IS NOT IRB APPROVED,
A MARK OF THE LEVEL OF MATURITY
FOR CERTAIN KINDS OF RESEARCH
STUDIES.
FOR OTHERS, THERE MAY BE
OPPORTUNITIES TO ENGAGE IN A
MUCH, MUCH EARLIER LEVEL, RIGHT?
SO WHERE ORGANIZATIONS MIGHT
WANT TO GET TO KNOW PEOPLE, GET
TO KNOW STORIES, LEARN FROM THE
COMMUNITY WHAT IT IS THEY DON'T
KNOW YET, QUITE ASIDE FROM ANY
PARTICULAR TECHNOLOGY OR TRIAL
JUST YET.
SO I THINK THAT THE CRITERIA
MIGHT BE THE WAY YOU POSITION
YOURSELF AND WHETHER YOU'RE
INVITED AS AN ORGANIZATION,
WHERE YOU CAN PRIORITIZE WHERE
YOU'RE BEING ASKED TO FULLY
PARTICIPATE AS A PARTNER, AS
OPPOSED TO ENDORSE, GIVE INPUT,
THOSE MIGHT BE THINGS THAT YOU
CAN CONSIDER.
ANOTHER ONE IS HOW DOES THIS
REALLY RETURN VALUE TO US?
IN A LOT OF CASES, INCREASINGLY,
SOME ENGAGEMENT REQUESTS ARE
GOING TO INVOLVE DATA, PATIENT
DATA, ANOTHER QUESTION YOU'VE
HAD.
>> DON'T GO THERE PLEASE!
>> RIGHT?
AND SO, AS ORGANIZATIONS YOU
HAVE RESOURCES.
YOUR RELATIONSHIP, YOUR
FAMILIES, YOUR PEOPLE, AND THEIR
INFORMATION IS VALUABLE.
YOUR RELATIONSHIPS ARE VALUABLE
ON A HUMAN LEVEL.
AND ALSO THIS DATA IS VERY, VERY
VALUABLE FROM A MONETARY
STANDPOINT.
AND SO THINKING ABOUT REALLY
CONSIDERING THAT YOU HAVE MUCH
MORE TO PROVIDE THAN JUST
PERSPECTIVES AND STORIES, THAT
THE INPUT AND PERSPECTIVES THAT
YOU BRING ARE POTENTIALLY GOING
TO MULTIPLY THE VALUE OF
WHATEVER IT IS THAT THEY ARE
DOING.
POSTURING IN A WAY THAT
CONSIDERING, OKAY, ARE WE BEING
RESPECTED PARTNERS, ARE WE BEING
LISTENED TO, AND THERE ARE
THINGS CAN YOU DO TO ESTABLISH
THAT.
THERE ARE KINDS OF DOCUMENTS,
BRIEFINGS, MATERIALS THAT SOME
ORGANIZATIONS HAVE DEVELOPED,
SAY, WHEN THEY GET APPROACHED BY
PHARMA OR INDUSTRY, TO SAY THESE
ARE THE GROUND RULES FOR HOW WE
WORK, THEY LEARN THESE THINGS
THROUGH HARD LINES.
MISSTEPS, BEING, TO A CERTAIN
EXTENT, MISTREATED BY THEIR
ENGAGEMENT PARTNERS.
SO RESPECT, POSTURING FOR
PARTNERSHIP, AND LISTENING, AND
CONSIDERING WHETHER THERE IS A
RETURN OF VALUE THAT DOESN'T
JUST HAVE TO BE IN MONETARY
COMPENSATION THAT COULD BE IN
TERMS OF HOW ARE YOU GOING TO
USE OUR INFORMATION, HOW WILL
THE PRIORITIES THAT WE INFORM,
HOW CAN WE EITHER MAKE SURE THAT
BENEFIT GETS BACK TO OUR
MEMBERSHIP OR HOW CAN WE
CONTINUE TO ENGAGE SUSTAINABLY
OVER TIME.
>> THAT WAS FANTASTIC.
JOE, YOU HAVE SOMETHING TO SAY?
I WAS GOING TO COME TO YOU NEXT
TO TALK ABOUT VALUE.
>> WELL, BESIDES OVERWHELMINGLY
AGREEING, ONE OF THE MOST
IMPORTANT THINGS OF MY
EXPERIENCE IS TOO FAR AWAY.
ONE OF THE THINGS THAT DOES
CREATE ISSUES IS NOT HAVING
THOSE RULES OF ENGAGEMENT LAID
OUT.
IT'S ONE OF MY ONGOING LARGEST
CRITICISMS OF ANYONE THAT WANTS
TO DO PATIENT ENGAGEMENT BECAUSE
VERY EASY TO WRITE A LINE IN A
PROJECT.
WE'LL ENGAGE PATIENTS, OR PUT IT
IN LEGISLATION, DROP IT IN.
BUT WHAT DOES THAT MEAN?
I THINK IT'S ALSO HELPFUL NOT
ONLY TO THE PERSON, THE COMPANY
CREATING AND COMING TO YOU, BUT
AS AN ORGANIZATION TO THINK WHEN
I SAY PARTNERSHIP, WE WANT TO BE
ENGAGED, THAT MEANS A SEAT AT
THE TABLE, AGAIN BEING ON THE
LEADERSHIP COMMITTEE, ON A
STEERING COMMITTEE, THAT DOES
NOT MEAN ENDORSEMENT OR MAYBE
THERE'S A PATHWAY TO
ENDORSEMENT, THINKING THROUGH
WHAT THAT LOOKS LIKE.
THE OTHER PIECE THAT I WANT TO
BRING UP CAME UP IN THE FIRST
PANEL AROUND CONTINUOUS NATURE
OF ENGAGEMENT AND IMPORTANCE
THERE.
IN MY DREAM AND PERFECT WORLD OF
PATIENT ENGAGEMENT THAT HAPPENS
ALONG A CONTINUUM THERE'S A
LEVEL OF CONTINUOUS ENGAGEMENT
WHERE YOUR PARTNERS ARE NOT
DROPPING IN FOR A QUICK PROJECT.
YOU DON'T HEAR FROM THEM FOR SIX
YEARS AND THEY POP BACK UP AGAIN
.
YOU HAVE ONGOING, WHETHER IT'S A
FORMALIZED PROJECT OR
RELATIONSHIP THAT SPANS THE
DURATION BUT, AGAIN, CAN HELP
REDUCE THAT BURDEN VERSUS
SOMEONE COMING OUT OF THE BLUE
TO SAY WE'D LIKE TO PARTNER,
LAST TIME YOU HAD DIFFERENT
LEADERSHIP TEAM AND A NEW
ENTITY.
SO MAINTAINING THOSE
RELATIONSHIPS ON THE FUNDER
SIDE.
>> GREAT.
THANK YOU.
I'M GOING TO COME TO COURTNEY.
I THREW OUT A GLIB COMMENT ABOUT
INDUSTRY FUNDING, EVERYTHING
THAT FNIH DOES, WHICH IS NOT
TRUE.
LOTS OF OTHER SOURCES OF
PHILANTHROPY, CORRECTING THE
RECORD.
MAYBE YOU CAN TALK MORE ABOUT
THE VALUE THE PATIENT
COMMUNITIES ARE BRINGING TO YOUR
WORK.
>> I'LL ANSWER THE QUESTION THAT
YOU THREW OUT, MAYBE IN A MORE
GENERAL WAY THAN YOU WOULD HAVE
HOPED.
BUT THE COMMENT THAT RACHEL MADE
ABOUT SOME PATIENT GROUPS NOT
GETTING ENGAGED UNTIL THERE'S AN
IRB APPROVAL, THAT WOULD
ELIMINATE ABOUT 99% OF THE
PATIENT ENGAGEMENT THAT THE FNIH
DOES BECAUSE WHEN WE ARE
THINKING ABOUT CLINICAL TRIAL
DESIGN WE'RE TALKING TO THE
PATIENT GROUPS FOR, YOU KNOW,
CLINICAL ASSESSMENT, HOW MANY IS
TOO MANY, WHAT'S USEFUL, WHAT'S
NOT USEFUL.
LONG-TERM FOLLOW-UP.
SO, FOR EXAMPLE, RIGHT NOW WE
ARE IN THE PROCESS OF FINALIZING
THE DESIGN OF A LONGITUDINAL
OBSERVATIONAL AND
BIOMARKER-BASED STUDY FOR
DEPRESSION.
AND MENTAL HEALTH OBSERVATIONAL
AND CLINICAL TRIALS HAVE
NOTORIOUS CHALLENGES IN LONG
TERM RETENTION AND FOLLOW-UP.
HEARING FROM THE PATIENTS
THROUGH MENTAL HEALTH AMERICA
AND THROUGH NOMI ABOUT WAYS WE
CAN LEARN FROM THEIR EXPERIENCE
TO HELP OVERCOME OR BE AWARE OF
CHALLENGES AS WE DESIGNING THE
CLINICAL TRIAL.
USING DATA, CONSENTING, ALL
THINGS IN THE DESIGN PHASE
BEFORE WE GET TO AN IRB OR AN
FDA APPLICATION.
SO, I THINK THAT'S SOMETHING TO
CONSIDER.
OBVIOUSLY IF YOU ARE LUCKY
ENOUGH TO HAVE PEOPLE BEATING
DOWN YOUR DOOR FOR PATIENT
ENGAGEMENT, THEN THERE'S CERTAIN
CONSIDERATIONS THAT GO ALONG
WITH THAT VOLUME, BUT KNOW THAT
THERE ARE ORGANIZATIONS THAT
REALLY DO VALUE YOUR INPUT FROM
THE IDEATION AND MOVING FORWARD
FROM THAT.
>> THAT'S GREAT.
SHEENA, SORRY.
I WAS THINKING ABOUT -- MY
FRIEND SHAYNA IS OVER THERE.
YOU SAID YOU WANTED TO TALK MORE
ABOUT THE VALUE AND IMPACT OF
THE WORK YOU'VE BEEN DOING.
>> YES, ABSOLUTELY.
TO YOUR QUESTION, I HAVE TO
AGREE WITH THE OTHER RESPONSES
REGARDING PRIORITIZATION AND
ADDING DOES A PUBLIC/PRIVATE
PARTNERSHIP ALIGN WITH YOUR
MISSION, WITH YOUR ORGANIZATION,
DO YOU HAVE SHARED GOALS?
OR IS THIS SOMETHING OUTSIDE OF
YOUR REALM THAT YOU'RE ABOUT TO
DRIVE INTO?
IS THERE A COMMUNITY, A SPACE
WHERE YOU CAN BUILD TRUST WITH
WHOEVER IS LEADING THAT?
IT TAKES TIME TO BUILD THAT
TRUST.
ARE YOU GOING TO DEVELOP THE
RELATIONSHIPS WITH WHOEVER IS
LEADING THESE PUBLIC/PRIVATE
PARTNERSHIPS?
I JUST WANTED TO SAY THAT AND I
AGREE WITH CLEAR
RESPONSIBILITIES AND ROLES AND
COMMITMENT FROM THE BEGINNING SO
EVERYONE IS ON BOARD WITH WHAT'S
HAPPENING FROM BOTH SIDES.
I THINK THAT JUST APPROACHES
THESE WITH HUMILITY AND
UNDERSTANDING WE'RE LEARNING
ALONG THE WAY, WILL BE HELPFUL.
BROADER IMPACTS, I DID WANT TO
MENTION, THIS IS REALLY IN
RESPONSE TO DR. BLACKSTONE'S
POINT ABOUT HOW CAN WE MOVE FROM
BASIC SCIENCE TO CLINICAL
OPPORTUNITIES, AND ONE THING
THAT NEURONEXT IS GROUND THE
WORK AND HOW DO WE GET THERE, IN
MULTIPLE WAYS.
FOSTERING THAT SUPPORT OF
COMMUNITY AND BIDIRECTIONAL
COMMUNICATION, NOT JUST OUR TEAM
SPEAKING WITH THE MEMBERS BUT
OUR MEMBERS, THEY HAVE A SEAT AT
THE TABLE ANYTIME WE HAVE A
MEMBERSHIP MEETING, NOT ON THE
SIDELINES, THEY ARE AT THE TABLE
WITH MEMBERS TO ACTIVELY
CONTRIBUTE AND THEY HAVE AN
OPPORTUNITY FOR SMALLER GROUP
CONVERSATIONS WITH FORUM MEMBERS
FOR TOPICS WE'RE DEVELOPING AS
WELL.
I THINK ANOTHER SIDE IS ALSO
WORKING WITH WITH OUR MEMBERS
CREATING IMPACTS, WE GET IN OUR
SPACES AND GROUP AND THINK IDEAS
ARE WONDERFUL, YOU WALK OUT THE
DOOR, THEY ARE LIKE WHAT ARE YOU
TALKING ABOUT?
NO IDEA.
THEY GROUND OR WORK.
IT'S A CRITICAL POINT.
WE HAD A FULL SESSION AROUND
HELPING TO COMMUNICATE AND
DISSEMINATE COMPLEX SCIENTIFIC
FINDINGS IN PLAIN LANGUAGE.
COMMUNICATION IS KEY.
IT'S CHALLENGING FROM A
SCIENTIFIC STANDPOINT.
HOW DO WE GET THAT MESSAGING OUT
TO THE PUBLIC AS WELL AS
INDIVIDUALS LIVING WITH THE
DISORDERS WE'RE STUDYING?
I THINK A BIG PART IS EVEN THE
TITLES OF CONSENSUS REPORTS, OF
OUR WORKSHOPS, DO YOU EVEN
UNDERSTAND WHAT WE'RE ADDRESSING
IN THIS MEETING?
NO?
OKAY, LET'S REVISE AND SHORTEN,
MAKE IT CRISPR.
THAT'S A REALLY CRITICAL POINT
THAT AS A COMMUNITY WE CAN
CONTINUE TO DO BETTER.
LASTLY LIKE HOW WE CONDUCT NOT
ONLY THE WORK OF OUR FORUM BUT
ACROSS THE ACADEMIES.
ONE MEMBER, J.R. RIZZO,
PROFESSOR AT NYU, HE DID HELP US
REVAMP ACCESSIBILITY PRODUCTS
AND HOW WE'RE THINKING ABOUT HOW
TO MAKE OUR MEETINGS MORE
ACCESSIBLE, WHETHER LANGUAGE,
WHETHER IT'S THE FORMAT AND
LAYOUT OF THE ROOM ITSELF SO
THAT WAS REALLY INFORMATIVE,
REALLY MOVING TOWARDS BROADER
IMPACTS THAT ARE HAPPENING NOW.
>> FANTASTIC.
ONE OF THE MOST IMPORTANT
POINTS, HAS TO BE MISSION
ALIGNED.
LET'S NOT FORGET THAT.
PEOPLE IN THIS ROOM DON'T HAVE
TIME OR MONEY TO WASTE, AND IT
CAN SEEM ENTICING IF INDUSTRY
SAYS THEY WANT TO GIVE YOU
MONEY, TO SAY YEAH, LET'S DO
THAT, DON'T BE AFRAID TO ASK FOR
THINGS IN RETURN, WHETHER THAT
IS IN FACT MONEY OR WHETHER IT'S
RETURN OF INFORMATION, RETURN OF
DATA, RETURN OF OTHER ASPECTS OF
THE RESEARCH PROCESS.
I DID WANT TO ASK, I WAS SO
INTERESTED THAT THE PREVIOUSLY
PANEL TALKED ABOUT COLLABORATION
THAN THE STATE OF THE SCIENCE,
THE STATE OF THE SCIENCE IS
AMAZING, SO MUCH GREAT SCIENCE,
AND AS YOU ARTICULATED WHERE WE
NEED HELP IS GETTING THE
SCIENCE, YOU KNOW, THROUGH THE
STAGE GATES AND INTO THE HANDS
OF PATIENTS AND PAID FOR.
BUT DO FOLKS FEEL LIKE THERE ARE
AREAS OF PARTICULAR OPPORTUNITY
IN NEUROSCIENCE RIGHT NOW WHERE
PARTICULARLY PEOPLE WORKING
ACROSS CONDITIONS MIGHT HAVE
OPPORTUNITY TO PARTNER AND
COLLABORATE, AND MAYBE I'LL
START WITH COURTNEY ACTUALLY.
>> YEAH, SO I TALKED A LITTLE
BIT ABOUT OUR ACCELERATED
MEDICINES PARTNERSHIP PROGRAM.
I HAD ACTUALLY JUST PULLED
PARTICIPANT NUMBERS FOR ANOTHER
PRESENTATION LAST WEEK AND THERE
ARE MORE PATIENT GROUPS INVOLVED
ACROSS THE 12 PROJECTS OF THAT
PROGRAM NOW THAN THERE ARE
INDUSTRY.
I THINK IT'S 42 INDUSTRY
PARTICIPANTS, AND 46 NON-PROFIT
ORGANIZATIONS.
SO THAT IS A SPACE THAT'S
CONTINUING TO GROW FOR US.
WE LAUNCHED TWO AMPs IN
NEUROSCIENCE LAST YEAR, ONE IS A
FOLLOW-ON EFFORT FROM OUR
PARKINSON'S DISEASE, SO
INCLUDING CONTINUING THE WORK OF
PARKINSON'S DISEASE AND OTHER
DISORDERS, AS WELL AS AMP AND
ALS WHICH WE GOT FROM IDEATION
TO EXECUTION IN RECORD TIME, YOU
KNOW, WITH THE PATIENT
COMMUNITIES IN MIND WHO DON'T
HAVE TIME TO WAIT FOR, YOU KNOW,
YEARS AND YEARS FOR A
PARTNERSHIP TO DEVELOP.
WE ALSO HAVE A PRETTY ROBUST
NEUROSCIENCE PORTFOLIO WITHIN
OUR BIOMARKERS CONSORTIUM.
SO WE HAVE REGULAR IDEATION
MEETINGS, HELPING TO IDENTIFY,
YOU KNOW, BIOMARKERS THAT THE
FIELD NEEDS TO MOVE THERAPEUTICS
FORWARD, AND PATIENT GROUPS AND
NON-PROFIT ORGANIZATIONS ARE
FREQUENTLY INVOLVED IN THOSE AS
WELL.
DEPRESSION INITIATIVE IS A BOARD
MEMBER, A NEW SPACE FOR US, OF
BUILDING SOMETHING OUTSIDE OF
ONE OF THE EXISTING GOVERNANCE
STRUCTURES.
AND WE HAVE NIMH SERVING IN AN
ADVISORY CAPACITY.
BUT AT THIS TIME THEY ARE NOT
PUTTING FUNDING INTO IT.
AND WE'VE HEARD FROM SOME PEOPLE
THAT DOESN'T COUNT AS A
PUBLIC/PRIVATE PARTNERSHIP IF
NIH -- WE OBVIOUSLY FEEL
DIFFERENTLY ABOUT THAT.
AND TAKE A BROAD PERSPECTIVE OF
PPP, NOT JUST DOLLARS.
WE'VE DONE A LITTLE BIT OF
EXPLORATION INTO WHAT A VENTURE
PHILANTHROPY FUND WOULD LOOK
LIKE, CERTAINLY NEUROSCIENCE IN
PARTICULAR, RARE DISEASES IN
NEUROSCIENCE ARE VERY
INTERESTING TO US IN THAT
RESPECT, AS WELL AS
UNDERRESOURCED AREAS.
>> THAT'S GREAT.
YOU'RE ON FIRM TERRITORY WITH
THE BROAD DEFINITION OF
PUBLIC/PRIVATE PARTNERSHIP, IT'S
AN IMPORTANT MESSAGE THE ASSETS
ARE NOT JUST FINANCIAL.
ANYBODY ELSE WANT TO SPECULATE
ABOUT AREAS OF OPPORTUNITY?
>> YEAH, THERE'S ONE THAT I
RECENTLY BECAME AWARE OF THAT
I'D LOVE TO SHARE, THE LIGHT
COLLECTIVE.
I THINK THE CATCH PHRASE IS NO
AGGREGATION WITHOUT
REPRESENTATION.
IF YOU'RE NOT AWARE OF HOW LIVED
EXPERIENCE DATA IS BECOMING MOD
--
COMMODITIZED, HAVING --
THERE'S -- I DIDN'T COUNT THEM,
MANY NON-PROFITS ARE PART OF
THAT.
THAT ORGANIZATION'S MISSION IS
TO PROTECT PATIENT DATA RIGHTS.
ALSO TO BROKER I THINK
COLLABORATIONS SO THAT THE
EXPERIENCE OF THESE DIFFERENT
INDIVIDUALS WHO MIGHT BELONG TO
AN ADVOCACY ORGANIZATION OR
PARTICULAR IDENTITY CAN BE
ENGAGED WITH AND CAN PARTNER AS
THESE OTHER TECHNOLOGIES ARE
DEVELOPED SO THEIR PERSPECTIVES
AND HOW DATA IS GETTING USED IS
BEING REPRESENTED.
I KNOW THERE'S DEFINITELY
PRODUCTS IN THE PARKINSON'S
DISEASE SPACE.
AS FAR AS I KNOW THE ONE THAT
I'M AWARE OF DOES A GREAT JOB
WITH DATA BUT I KNOW THEY HAVE
ALSO HAD A VERY LOT OF PATIENT
ENGAGEMENT AS THEY HAVE BEEN
DEVELOPING THEIR TECHNOLOGIES SO
THERE MAY BE MORE OPPORTUNITIES
EITHER JUST JOINING IN A
COLLABORATIVE AND BEING PART OF
THE WORK THEY ARE DOING OR
POSSIBLY BEING CONNECTED THROUGH
THEM WITH SOME OTHER PROJECTS.
THAT'S A VERY COOL INITIATIVE.
THE LIGHT COLLECTIVE, ANDREA
DOWNING IS THE PERSON BEHIND
THAT.
>> I'M PRIMING THE AUDIENCE.
TELL US ABOUT YOUR PARTNERSHIP
EXPERIENCES, GOOD, BAD,
INDIFFERENT.
I'LL START CALLING ON PEOPLE IF
YOU DON'T.
THEY ARE ASLEEP AT 2:10 IN THE
AFTERNOON.
SOMEBODY MENTIONED VENTURE
PHILANTHROPY TOPIC, WAS THAT
COURTNEY?
SHOULD WE GO THERE?
DO WE WANT TO TALK ABOUT VENTURE
PHILANTHROPY AND WHAT ITS
UTILITY IS AND WHO SHOULD THINK
ABOUT IT AND WHAT THEY SHOULD
EXPECT?
>> SO WE DID ABOUT -- JUST ABOUT
A SIX-MONTH EXPLORATION IN
CONJUNCTION WITH A TEAM FROM
McKENZIE, WHAT COULD A VENTURE
FRONT FUND LOOK LIKE FOR THE
FNIH, IS IT SOMETHING THAT WOULD
BE CLOSER TO THE VENTURE END OF
VENTURE PHILANTHROPY, SOMETHING
CLOSER TO THE PHILANTHROPY END
OF VENTURE PHILANTHROPY?
WE ALSO EXPLORED KIND OF WHERE
IN THE PIPELINE WOULD IT BE MOST
ADVANTAGEOUS FOR FNIH TO BE
ENGAGED, IS IT SOMETHING THAT'S
VERY EARLY ON HELPING TO SUPPORT
PROMISING BASIC SCIENCE THAT
MIGHT BECOME COMMERCIALIZABLE
DOWN THE ROAD, SOMETHING ON THE
TAIL END?
WE HAD CONVERSATION WAS SBIR
PROGRAM OFFICERS, SMALL BUSINESS
INNOVATION RESEARCH PROGRAM, AT
A NUMBER OF I.C.s IN THE NIH
INCLUDING NINDS, AND, YOU KNOW,
TO SEE IF THERE ARE
OPPORTUNITIES TO KIND OF HELP
BRIDGE COMPANIES OUT OF THE SBIR
PROGRAM WHO ARE MAKING REALLY
GREAT PROGRESS BUT MAYBE AREN'T
QUITE READY FOR MORE TRADITIONAL
INVESTMENT.
ONE OF THE CHALLENGES FOR US WAS
ARTICULATING WHAT DO WE BRING TO
THE TABLE?
WHAT IS UNIQUE ABOUT FNIH THAT
WOULD SET US APART FROM THE
OTHER THOUSAND LIFE SCIENCE
INVESTMENT AND VENTURE FUNDS.
AND HEARKENING BACK TO SOMETHING
THAT I SAID AT THE BEGINNING,
THE FACT THAT WE DON'T HAVE A
SPECIFIC DISEASE OR HEALTH AREA
OF FOCUS IS ACTUALLY QUITE A
CHALLENGE FOR US IN THE VENTURE
SPACE BECAUSE UNLIKE THE T1D
FOUNDATION THAT INVESTS IN
PROMPTING T1D TECHNOLOGIES OR I
THINK THERE'S ALSO ONE WITH THE
AMERICAN HEART ASSOCIATION AND
AMERICAN CANCER ASSOCIATION.
WE HAVE THE WHOLE RANGE OF HUMAN
HEALTH IN FRONT US, AND ALL OF
THE WORK THAT THE NIH IS DOING.
AND SO KIND OF THE -- WE DIDN'T
LAND ON MOVING FORWARD WITH
VENTURE PHILANTHROPY FUND YET,
BUT I THINK THE TWO THINGS WE
DID ALIGN ON, ONE IS THAT WE
WANTED TO LOOK FOR AREAS OF
HUMAN HEALTH THAT DON'T RECEIVE
AS MUCH TRADITIONAL VENTURE
FUNDING, SO RARE DISEASE SPACES,
PEDIATRICS, WOMEN'S HEALTH,
GLOBAL HEALTH, INFECTIOUS
DISEASE, SO FORTH, BECAUSE
THAT'S CLEARLY A WHITE SPACE
AVAILABLE.
THE OTHER IS WE HAVE A VALUE
PROPOSITION IN OUR CONNECTION TO
NIH THAT REALLY NO OTHER
ORGANIZATION CAN CLAIM IN THE
SAME WAY.
AND SO LOOKING FOR OPPORTUNITIES
TO EITHER WORK WITH NIH
TECHNOLOGIES, TECHNOLOGIES THAT
NIH HAS FUNDED, HAVING THAT
RELATIONSHIP BETWEEN THE NIH AND
FNIH IS SOMETHING THAT COULD
POTENTIALLY SET US APART IN AN
OVERSATURATED FIELD OF
INVESTMENT.
>> ABSOLUTELY FAIR.
DIVERSE DEAL FLOW.
WE'VE GOT A QUESTION.
>> IS THAT MY CUE?
>> I DIDN'T EVEN HAVE TO CALL ON
YOU.
>> ERIC COLE, DANNY WALKER
ALLIANCE, MANY OF US REPRESENT
GLOBAL CONSTITUENCIES OF
PATIENTS.
WE FIND OURSELVES IN THIS SPACE,
LIKE THE HYDROCEPHALUS
FOUNDATION AND BAND TOGETHER.
YOU JUST MENTIONED A COUPLE
THINGS, DR. SILVERTHORN.
AS RELATES TO VENTURE
PHILANTHROPY, ARE THERE
OPPORTUNITIES TO LOOK
INTERNATIONALLY FOR THAT?
AND PERHAPS COME TOGETHER IN A
PARTNERSHIP AND THEN PARTNER
WITH SOME I.C.s AT NIH THROUGH
CRADAs SO WE CAN BE ABLE TO
PERHAPS BRING A NEW APPROACH TO
FUNDING THINGS SPECIFICALLY FOR
RARE DISEASE?
>> I SAW AN ARTICLE LAST WEEK
ABOUT A HARVARD RESEARCHER THAT
GOT A MULTI-MILLION DOLLAR
INVESTMENT FROM A VENTURE FIRM
IN THE MIDDLE EAST.
WHICH IS A PRETTY NOVEL WAY TO
FUND YOUR RESEARCH.
AND I THINK THAT THERE ARE LOTS
OF OPPORTUNITIES THAT WILL
PROBABLY COME FORWARD IN THE
FUTURE.
THE INTERNATIONAL QUESTION FOR
US HAS BEEN CHALLENGING.
SO WITHIN THE BESPOKE GENE
THERAPY CONSORTIUM FOR EXAMPLE
BECAUSE OF THE DIFFERENCES IN
REGULATORY ENVIRONMENTS BETWEEN
THE U.S. AND OUTSIDE OF THE
U.S., WE HAD TO MAKE THE
DECISION TO FOCUS WITHIN U.S.,
BUT WE ARE DOING WHATEVER WE CAN
TO HAVE COORDINATING
CONVERSATION WAS OTHER
REGULATORY BODIES.
BUT WE KNOW THAT RARE DISEASES,
DISEASES THAT ARE RARE IN THE
U.S., ARE NOT NECESSARILY RARE
PARTICULARLY FROM AN INVESTMENT
PERSPECTIVE WHEN YOU LOOK AT
GLOBAL POPULATIONS.
AND SO ANYTHING THAT CAN BE DONE
TO BRING TOGETHER SOME OF THOSE
INTERNATIONAL COMMUNITIES I
THINK REALLY DOES HELP MAKE IT
MORE FEASIBLE FOR THINGS TO MOVE
FORWARD INTO CLINICAL TRIALS AND
INTO PRODUCTS.
>> THAT'S GREAT.
THERE ARE A LOT OF THINGS THAT
FOUNDATIONS CAN FUND TOO, RIGHT?
OTHER THAN BASIC ACADEMIC
GRANTS, AND MAYBE SHORT OF
ACTUALLY MAKING EQUITY
INVESTMENTS IN COMPANIES AND DR.
LODISH CITED EARLIER CROs,
THERE ARE A NUMBER OF
FOUNDATIONS THAT WILL ACTIVELY
LOOK AT THEIR BASIC GRANTS
PORTFOLIO AND SAY THIS SEEMS
LIKE IT'S READY TO PULL THROUGH
INTO, YOU KNOW, INTO
PRE-CLINICAL WORK AND THERE'S A
DEARTH OF FUNDING AND INTEREST
IN DOING THAT WORK IN AN
ACADEMIC SETTING, COMPANIES
DON'T WANT TO SEE IT YET.
SO SOME FOUNDATIONS WILL BE LIKE
WE'RE GOING TO CONTRACT TO A CRO
TO DO INTERMEDIATE STEPS AND IT
CAN GO BACK TO THE ORIGINAL
INVESTIGATOR IF THEY HAVE THE
APPETITE.
MAYBE THEY CAN ENGINEER A
HANDOFF TO A BIOTECH THAT MIGHT
BE INTERESTED.
THERE ARE A LOT OF THINGS
FOUNDATIONS CAN DEPLOY FUNDS
AGAINST, EVERYBODY KNOWS SETTING
UP A REGISTRY, VALUABLE
SOURCINGS OF DATA FOR
STAKEHOLDERS.
I KNOW PEOPLE IN THE ROOM HAVE
AMAZING PARTNERSHIPS, AND I'M
GOING TO ASK SOME TO TALK ABOUT
THEM.
I'LL TELL YOU WHO I HAVE IN MIND
AND YOU CAN DECIDE IF YOU WANT
TO TALK ABOUT IT OR NOT.
I DID TELL RON BARTEK I MIGHT
ASK HIM TO TALK ABOUT THE ROLE
OF THE NATURAL HISTORY DATA YOU
COLLECTED IN THE SKY CLARIS
APPROVAL, IT'S A TWIST.
RICH BRENNAN, CMS.
SHAYNA, THE WORK AFTD DOES WITH
COMPANIES, BRINGING THEM
TOGETHER IN A COLLECTIVE.
>> I'VE BEEN VOLUNTOLD TO TALK.
IT'S TEMPTING.
I COULD GO ON FOR A COUPLE
HOURS, TO ANSWER KRISTIN'S
ENTREATY.
I'LL CUT IT SHORT AND FOCUS ON
THE TOPIC THAT YOU MENTIONED,
IMPORTANCE OF OUR NATURAL
HISTORY STUDY.
WE FOUND -- MY WIFE AND I ARE
CO-FOUNDERS, FOUNDED 26 YEARS
AGO, WE KNEW WE WANTED TO FOCUS
ON RESEARCH, THERE WERE OTHER
THINGS BUT WE WANTED TO MAKE OUR
MARK IN HELPING MOVE THE
RESEARCH FORWARD.
WE KNEW THAT TO DEVELOP THE KIND
OF PUBLIC/PRIVATE PARTNERSHIPS
THAT WE'RE ALL TALKING ABOUT
THAT WOULD INCLUDE OUR PATIENT
FAMILIES, INDUSTRY PARTNERS, OUR
SCIENTIFIC INVESTIGATORS,
GOVERNMENT PARTNERS LIKE THE NIH
AND FDA, AND OTHER PATIENT
ORGANIZATIONS ESPECIALLY THE
UMBRELLA GROUPS LIKE
FASTERCURES, EVERY LIGHT
FOUNDATION, WE'RE ALL IN THIS
TOGETHER.
SORRY.
THAT WE WOULD NEED TO DEVELOP
ASSETS THESE PARTNERS WOULD NEED
TO SUCCEED.
THE ASSETS THAT OUR INDUSTRY
PARTNERS ESPECIALLY WOULD NEED,
LIKE WE NEED TO CHARACTERIZE THE
DISEASE, TO FUND THE BASIC
SCIENCE PROJECTS THAT WOULD
CHARACTERIZE OUR DISEASE, THAT
WOULD GIVE THESE INDUSTRY
PARTNERS AN IDEA WHAT OUR
DISEASE IS, WHAT CAUSES IT.
WE WOULD NEED TRANSLATIONAL
SCIENCES, LIKE CELLS OF THE
ANIMALS, SO FORTH, THE
BIOMARKERS.
THIRD WE COULD NEED CLINICAL
ASSETS LIKE OUR NATURAL HISTORY
STUDY, PATIENT REGISTRY TO
RECRUIT CLINICAL TRIALS.
AND A CLINICAL NETWORK, ALL OF
WHICH WE'VE DEVELOPED OVER TIME.
TO COME TO THE POINT KRISTIN
ASKED ME ABOUT, THAT NATURAL
HISTORY STUDY, WON'T GO INTO
DETAILS HOW WE ACCIDENTALLY
STARTED IT, BUT IT WAS VERY
FORTUITOUS.
IT'S BEEN GOING ON FOR MORE THAN
20 YEARS.
AND SO WE COME TO 19 -- TO 2022,
AND WE HAVE A SMALL BIOTECH IN
AUSTIN, TEXAS, THAT'S GOT THIS
TRUCK -- DRUG WE HELPED THEM
WORK ON, THEY HAVE DONE A PHASE
2 CLINICAL TRIAL THAT SHOWED WE
THOUGHT THAT IT SLOWED
PROGRESSION OF OUR DISEASE,
THAT'S RELENTLESSLY PROGRESSING.
AND WE WENT WITH THEM TO THE
FDA.
FDA SAID THESE DATA ARE
INTERESTING BUT NOT SUFFICIENTLY
PERSUASIVE, DO ANOTHER CLINICAL
TRIAL.
WE USED UP MOST OF THE PATIENTS
THAT WOULD BE ELIGIBLE AND KNEW
WE COULDN'T.
WE WORKED INSTEAD WITH A
COMPANY, GAVE FULL ACCESS TO
NATURAL HISTORY DATABASE.
THEY CONSTRUCTED THREE NEW
DATASETS TO SUPPLEMENT THEIR NEW
DRUG APPLICATION.
ONE OF THEM WAS THE USE OF OUR
NATURAL HISTORY DATA AS AN
EXTERNAL COMPARATOR TO THE
CLINICAL TRIAL DATA, ESPECIALLY
IN THE OPEN LABEL EXTENSION FOR
3 YEARS.
THAT COMPARISON DEMONSTRATED
THAT THE CAREFULLY MATCHED
PATIENTS FROM OUR NATURAL
HISTORY STUDY TO THOSE 136 IN
OPEN LABEL EXTENSION, SO THE
ONES FROM NATURAL HISTORY
OBVIOUSLY NOT ON ANY DRUG, THERE
WASN'T ONE.
THE ONES IN THE OPEN LABEL
EXTENSION ALL ON DRUG ENJOYED
55% SLOWING IN PROGRESSION OVER
THE 3-YEAR PERIOD, WENT BACK TO
PHASE 2 CLINICAL TRIAL IT WOULD
BE 70% SLOWING OF PROGRESSION.
THAT CONVINCED THE FDA, SO ON
RARE DISEASE DAY OF 2023 THEY
ANNOUNCED THE FIRST APPROVAL FOR
ATAXIA.
NOW I WANT TO CELEBRATE THE
75th ANNIVERSARY OF NINDS BY
TELLING YOU A QUICK STORY.
OUR SON KEITH WAS DIAGNOSED 27
YEARS AGO, AGE 11.
MY WIFE RACHEL WAS WORKING FOR A
LOUISIANA CONGRESSMAN WHO WAS
ASKED BY ONE OF HIS COMMITTEE
MEMBERS TO GIVE THE KEYNOTE
SPEECH AT A GALA FOR A DIFFERENT
ATTACKSIA.
BILLY TURNS TO RACHEL, YOU KNOW
MORE, COULD YOU WRITE MY SPEECH?
RACHEL AND I WROTE HIS
PRESENTATION FOR THE KEYNOTE.
RACHEL GOES TO THE GALA.
SITS AT THE HEAD TABLE WITH THE
CONGRESSMAN BILLY TOZAN, BILLY
GIVES A SPEECH, PEOPLE START
BREAKING UP.
A WOMAN ALSO AT THE HEAD TABLE
THAT WE DIDN'T KNOW COMES AROUND
TO RACHEL AND SAYS, SO WHY ARE
YOU HERE?
I WROTE THE CONGRESSMAN'S
SPEECH.
WHY SO?
MY SON HAS A DIFFERENT ATAXIA,
WHICH ONE?
SHE SAYS, I'M THE PROGRAM
OFFICER AT NINDS, FOR
NEURODEGENERATIVE DISEASES, I'D
LIKE TO INVITE YOU AND YOUR
HUSBAND TO COME OUT AND HAVE
BREAKFAST WITH ME THURSDAY.
AT NINDS.
WE DID THAT.
SHE SAID AFTER BREAKFAST I'M
GOING TO TAKE YOU TO THE
NATIONAL ADVISORY COUNCIL
MEETING.
THIS IS A PLUG.
HERE WE ARE AT THE NON-PROFIT
FORUM.
A WONDERFUL FORUM THAT WALTER
SAYS WE'RE GOING TO EVEN DO
BETTER THIS YEAR THAN LAST YEAR.
HE'S ALWAYS RIGHT.
SO THE OTHER FORUM THAT I'M
GOING TO INVITE, IF YOU'RE NOT
ALREADY INVOLVED, IS THE
NATIONAL ADVISORY COUNCIL MEETS
THREE TIMES A YEAR, SO WE WENT
UP TO THAT MEETING, THAT
THURSDAY MORNING, AFTER
BREAKFAST.
WE GOT TO LEARN HOW THE NINDS
WORKS AND WHY.
AND WE GOT DEEPLY INVOLVED.
AND THE PROGRAM OFFICER CAME UP
TO ME AFTER THAT AND SAID, NOW I
WANT TO ASK YOU IF YOU CAN HELP
US DEVELOP TWO-WAY
COMMUNICATIONS BETWEEN OUR NINDS
PERSONNEL AND YOUR SCIENTISTS.
WE DON'T HAVE THAT.
WE WANT TO BE ABLE TO
COMMUNICATE WITH THEM.
WE WANT THEM TO BE ABLE TO
COMMUNICATE WITH US, WE THINK
YOU SHOULD DEVELOP AN
ORGANIZATION.
WE HAD ALREADY DECIDED TO
DEVELOP THE ORGANIZATION BUT WE
DIDN'T TELL JOVANA THAT.
FAST FORWARD A FEW MONTHS, I WAS
ASKED TO SERVE ON THE NATIONAL
ADVISORY COUNCIL, WHICH I WAS
ABLE TO DO FOR 4 YEARS.
IT'S A WONDERFUL FORUM.
YOU GET TO KNOW YOUR PROGRAM
OFFICERS VERY WELL.
YOU HAD THE OPPORTUNITY EARLIER
TODAY TO MEET WITH YOUR PROGRAM
OFFICERS.
I WOULD ENCOURAGE YOU TO
CONTINUE THAT CONVERSATION, AND
YOU CAN PARTICIPATE IN THE
NATIONAL ADVISORY COUNCIL
MEETINGS VIRTUALLY OR WE'RE BACK
TO DOING THEM IN PERSON, SO
RIGHT HERE IN THIS ROOM.
I WOULD ENCOURAGE YOU TO GET
INVOLVED WITH THESE WONDERFUL
PEOPLE AT NINDS.
THANK YOU.
>> THANK YOU FOR SHARING YOUR
STORY.
>> IT'S ALWAYS HARD TO FOLLOW
RON.
I'M RICH BRENNAN, ALS
ASSOCIATION.
THANK YOU FOR PUTTING THIS ON
FOR THE ORGANIZATIONS INVOLVED.
WE JUST FINISHED A TWO-YEAR
ENGAGEMENT WITH NATIONAL
ACADEMIES, ALS, SORT OF HOW TO
MAKE A DIFFERENCE IN THE
RESEARCH PORTFOLIO FOR ALS, HOW
TO MAKE A CLINICAL DIFFERENCE
FOR PEOPLE WITH ALS.
COURTNEY, WE'VE PARTICIPATED IN
AMP ALS, THERE'S A NUMBER OF
PUBLIC/PRIVATE PARTNERSHIPS IN
THE ALS SPACE CRITICAL TO OUR
WORK.
NINDS, AMAZING AS FAR AS THEIR
WORK FOR US IN ADVANCING THE
RESEARCH
RESEARCH BEHIND ALS.
THANK YOU, WALTER.
WITH FDA WE'RE PART OF THAT, THE
I GUESS CONSORTIUM WHERE YOU'RE
ALLOWED TO CONNECT WITH, YOU
KNOW, EARLY DEVELOPERS OF NEW
TECHNOLOGIES AND HAVE
CONVERSATIONS, TO MAKE THINGS
REGULATORY READY.
SO I HAVE, YOU KNOW, SPENT TIME,
ENERGY, BLOOD, SWEAT AND TEARS
IN ALL THESE PLACES, IN MY ROLE.
I WOULD SAY I WAS UP AT
BIOINTERNATIONAL CONFERENCE LAT
WEEK AND SPOKE ON A PANEL ABOUT
THE WHY OF RESEARCH, WHY
RESEARCH IS BECAUSE WE CAN USE
BIOMEDICAL SCIENCE TO MAKE A
DIFFERENCE IN THE LIVES OF
PEOPLE.
AND THAT THERE'S A TREMENDOUS
AMOUNT OF INVESTMENT AND
PARTNERSHIP BEHIND THAT.
IT'S CRITICAL, THE FACT THAT WE
IDENTIFY RESOURCES AND
PARTNERSHIPS, AND SO THANK YOU
FOR FOCUSING ON THIS.
MY QUESTION FOR THE PANEL IS
REALLY WHAT I'VE SEEN IS THAT IT
IS GREAT TO ENGAGE WITH NATIONAL
ACADEMIES, WITH THE FNIH, OR
WITH OTHERS, EVEN THE BRAIN
COALITION GOT US CONNECTED TO
THE PARTNERSHIP UP FDA, IT'S
MORE OF THE HANDOFFS.
HOW DO WE TAKE THE NATIONAL
ACADEMIES' WONDERFUL REPORT OVER
A 2-YEAR PERIOD THAT HAS A
NUMBER OF AWESOME WONDERFUL
THINGS THAT WE NEED TO BE ABLE
TO ACTIVATE AS A COMMUNITY, AND
MAKE THAT HAPPEN.
HOW DO WE GET THAT TO THE NEXT
STEPS?
THAT'S PART OF IT.
I ALSO WANT TO GET BACK TO THE
POINT WHERE I MADE BACK IN BIO,
I BELIEVED THAT WE WERE ON THE
PRECIPICE OF OPENING THE
FLOODGATES OF HAVING PATIENTS
INVOLVED IN ALL ASPECTS OF
RESEARCH, IN THE DEVELOPMENT OF
THESE NEW INNOVATIONS.
MY QUESTION IS WHAT ARE BEST
PRACTICES BEHIND THAT, HOW ARE
YOUR ORGANIZATIONS WORKING ON
THOSE THINGS?
>> I THINK THAT MIGHT BE MY
CLOSING QUESTION, WE'RE AT THE
END OF THE SESSION.
WE'LL DO A QUICK ROUND STARTING
WITH SHEENA, TO ADDRESS IN A
MINUTE, HOW DO YOU PULL THROUGH
ACROSS THE CONTINUUM OF PRODUCT
RESEARCH AND DEVELOPMENT, THESE
PRIORITIES OF PATIENTS, INSIGHTS
OF PATIENTS, ENGAGEMENT AND
INSIGHTS TO SHARE IN A MINUTE OR
LESS.
>> YEAH, THANK YOU FOR YOUR
SUPPORT.
WE CAN CREATE SPACES TO HELP
BRING IMPLEMENTERS TOGETHER, TO
COORDINATE, I WOULD LOVE TO CHAT
AFTER THIS.
TO THE QUESTION AROUND, YOU
KNOW, THOSE IN THIS ROOM LIKE I
THINK THAT WE THINK ABOUT THESE
PUBLIC/PRIVATE PARTNERSHIPS, I
KEEP GOING BACK TO THE EXAMPLE
OF PROJECTS IN COLLEGE,
SOMETIMES IT'S EASIER TO DO IT
ALONE BUT YOU'RE MISSING OUT ON
RESOURCES FROM OTHER PEOPLE TO
COME TOGETHER TO HELP
POTENTIALLY BRING FORTH
SOMETHING OF GREATER IMPACT,
LEVERAGING EACH OTHER'S
STRENGTHS, AREAS WE CAN IMPROVE
ON.
I THINK IN TERMS OF ADDRESSING
THIS, I THINK MOVING FORWARD,
IT'S REALLY ESSENTIAL THAT LIVED
EXPERIENCE REMAINS THAT CATALYST
TO HELP DRIVE REALLY URGENCY AND
RELEVANCY OF THE WORK THAT WE DO
BUT IT'S NOT YOU ALONE.
EVERYONE HAS TO BE ACCOUNTABLE
AND STEP UP TO DRIVE THESE
EFFORTS.
MY THING, MY ONE-MINUTE THING,
MAKING SURE WE'RE ALL PUTTING
OUR SELF FORWARD TO DRIVE
PUBLIC/PRIVATE PARTNERSHIPS.
THERE'S A LOT OF PRESSURE ON THE
PATIENT COMMUNITIES AND PATIENT
ORGANIZATIONS, PEOPLE WITH LIVED
EXPERIENCES, WE ALL CAN HAVE
OUR -- PUT OUR SELF FORWARD TO
DRIVE THE EFFORTS FORWARD.
>> FIRST THING THAT CAME TO MIND
WE NEED CHAMPIONS AND
COMMUNICATORS.
THE PUBLIC/PRIVATE PARTNERSHIPS
THAT FNIH BUILDS AND EXECUTES
DON'T DIRECTLY CREATE THE
THERAPIES OR THE TREATMENTS OR
THE DEVICES.
THEY ENABLE OTHERS TO DO THAT
WORK.
AND OUR PARTNERS, OUR PATIENT
COMMUNITIES, OUR ADVOCATES ARE
THE ONES WHO HELP US DISSEMINATE
THOSE RESOURCES AND MAKE SURE
THE WORK DONE IN THE PARTNERSHIP
ENDS UP HAVING VALUE TO THE
PATIENTS AND SO WE RELY ON OUR
OWN COMMUNICATIONS EFFORTS, WE
HAVE A GROUP TO TELL THE STORY
OF WHAT FNIH IS ACCOMPLISHING.
NIH IS A GREAT CHAMPION.
I WANT TO FOLLOW ON THE THREAD
OF KUDOS TO NINDS BECAUSE YOU
MIGHT NOT KNOW THAT IN ADDITION
TO PUBLIC/PRIVATE PARTNERSHIPS
THE FNIH HAS AN AWARDS PROGRAM
AND ONE OF THE AWARDS WE GIVE
OUT EVERY YEAR IS THE CHARLES
SANDERS PARTNERSHIP AWARD, AND
NINDS WAS LAST YEAR'S WINNER FOR
THEIR EXORBITANT SUPPORT,
ENGAGEMENT, AND CHAMPIONING OF
PUBLIC/PRIVATE PARTNERSHIPS.
>> SITTING IN THE RIGHT PLACE.
>> A FEW THOUGHTS.
I'VE ALWAYS BOUGHT INTO THE
CONCEPTS OF BUILDING THE BIGGEST
TENT.
SO AS YOU'RE PLANNING YOUR
INITIAL PHASE, THINKING ABOUT
THE CONTEXT OF A GROUP WE'VE
BEEN WORKING WITH, PART OF THAT
EARLY STAGE STRATEGY WAS, OKAY,
WHAT HAPPENS WHEN WE'RE
SUCCESSFUL HERE?
WHERE DO WE GO FROM HERE?
HOW WILL WE MOVE INTO WHAT IS
ULTIMATELY NOW PHASE 2 OF THIS
WORK?
AND, AGAIN, THAT'S BEEN
INCREDIBLY HELPFUL.
TO THE SECOND QUESTION,
CONTINUOUS ENGAGEMENT, AGAIN A
PASSION OF MINE.
WE HAVE METHODOLOGY.
WE HAVE THE ABILITY TO DO IT.
I THINK THE PIECE THAT WE'RE
STARTING TO SEE MORE ON THIS IS
SHOWING HOW PATIENT INPUT IS
CHANGING DECISIONS.
THAT GOES TO TALKING ABOUT THE
VENTURE CAPITAL SPACE MAKING AN
INVESTMENT DECISION BASED DOWN
MET NEEDS, BASE ON PATIENT
PERSPECTIVE, LOWEST-LAUNCH TRIAL
DESIGN, PATIENT JOURNEY, ALL
LEVELS OF RESEARCH.
WHEN YOU'RE MAKING CHANGES
BECAUSE YOU'VE GOT INPUT FROM
PATIENTS THAT NEEDS TO BE
DOCUMENTED.
IF YOU'RE UNABLE TO MAKE
CHANGES, TO DOCUMENT THAT.
WHAT CHANGED, WHAT DIDN'T,
CONTINUING TO SHOW THAT IMPACT
IN A WAY THAT DEMONSTRATES THAT
THINGS ARE CHANGING BECAUSE
PATIENTS HAVE A VOICE.
>> I'LL JUST ANSWER FROM A
COMPLEMENTARY PERSPECTIVE
ALTHOUGH I AGREE WITH ANSWERS
GIVEN.
I THINK WHEN IT COMES TO
CARRYING THINGS THROUGH AND
ACTUALIZING RECOMMENDATIONS, AND
OTHER THINGS, WHEN IT DOESN'T
HAPPEN THERE'S A CAPABILITY
THAT'S MISSING OR THERE'S A
VALUE MISALIGNMENT OR INCENTIVE
THAT'S NOT THERE.
SO I THINK WITH SOME OF THE
THINGS THAT ARE RECOMMENDED FOR
THEM TO HAPPEN THEY OFTEN
INVOLVE PLAYERS, MULTIPLE
PLAYERS, ACROSS A COMPLEX
ECOSYSTEM.
AND SO IDENTIFYING WHO HAS AN
INTEREST IN THAT TO BEGIN WITH,
AND CREATIVELY COLLABORATING TO
EITHER ALIGN INCENTIVES OR
CREATE SITUATIONS WHERE THERE
CAN BE VALUE, IT'S POSSIBLE
THERE'S NOT RESOURCES HERE TO
DEVELOP THAT CAPABILITY BUT
MAYBE THIS OTHER ORGANIZATION
CAN, OR THIS OTHER PART OF THE
SYSTEM ECOSYSTEM CAN.
WITH COLLABORATIVE SPACES,
EITHER INDUSTRY GROUPS,
COLLABORATIVE COMMUNITIES LIKE
WHAT FDA HAS, THESE -- AND ANY
KIND OF MICROCOSM OF THAT THAT'S
PROJECT BASED, I THINK THERE'S
ROOM TO GET CREATIVE WHEN WE'RE
WORKING TOGETHER, ESPECIALLY
WHEN WE HAVE MEMBERSHIPS ACROSS
SECTORS, MAKING SOME OF THOSE
THINGS HAPPEN, JUST A MATTER OF
WHY ISN'T IT HAPPENING.
>> FANTASTIC.
SO I THINK IN CLOSING I'LL
SAYs I HOPE FOLKS HAVE COME TO
UNDERSTAND PATIENT ORGANIZATIONS
HAVE A LOT OF ASSETS AND
CAPABILITIES THAT ARE CRITICAL
TO THIS PROCESS OF RESEARCH AND
PRODUCT DEVELOPMENT THAT THEY
ARE VITAL PARTNERS.
YOU DON'T HAVE TO HAVE A LOT OF
MONEY.
YOU DO HAVE TO HAVE A LOT OF
WILLPOWER AND SHOULDN'T BE
AFRAID TO ASK TO BE EQUAL
PARTNERS WITH OTHER STAKEHOLDERS
IN THIS PROCESS.
I'LL TURN THE BATON BACK OVER --
JOIN ME IN THANKING THIS
FANTASTIC PANEL OF SPEAKERS.
>> THANK YOU.
WE'VE BEEN HEARING A LOT OF
WONDERFUL STORIES THROUGH THE
DAY.
OUR LAST SESSION IS FITTINGLY
STORYTELLING, WE HAVE A BRIEF
BREAK BUT PLEASE COME BACK IN
TEN MINUTES, 2:45.
SNACKS IN THE NEXT ROOM OVER.
LET'S ALL COME BACK AND HEAR
ABOUT THE POWER OF STORYTELLING.
>> WE HAVE A GREAT PANEL TO TALK
ABOUT STORYTELLING.
I'LL TURN IT OVER TO DR.
KOROSHETZ.
>> OKAY, FOLKS.
YEAH, WE'RE REALLY EXCITED ABOUT
OUR FINAL PANEL TO TAKE US HOME.
WE ALWAYS END THE NON-PROFIT
FORUM WITH STORIES FROM OUR
NON-PROFIT GROUPS.
STORIES THAT HOPEFULLY CAN
MOTIVATE AND HAVE LESSONS FOR
THE REST OF US.
AND I WANT TO RECOGNIZE SUNITHA
MALPATI TO CHAIR THE SESSION.
>> THANK YOU FOR STICKING WITH
US THIS LAST SESSION.
WE SPENT THE DAY I THINK TALKING
ABOUT THE INNOVATIONS AND
CHALLENGES AND OPPORTUNITIES IN
NEUROSCIENCE AND RESEARCH.
I WOULD LIKE TO SPEND THIS
SESSION ON SOMETHING THAT IS
CRITICALLY IMPORTANT,
STRATEGICALLY IMPORTANT TO
MOVING THE NEEDLE ON SCIENCE.
WE THINK OF IT AS A SOFT SKILL,
SOFT TOOL, THAT IS STORYTELLING.
EVERYONE IN THE ROOM HAS A STORY
TO TELL ABOUT WHAT BROUGHT THEM
HERE AND PROBABLY INCREDIBLE
STORIES THAT HAVE MOVED THE
NEEDLE IN SCIENCE.
WE WOULD LOVE TO ENGAGE YOU IN
THE SECOND HALF OF THE SESSION
AND BRING YOUR STORIES ABOUT THE
POWER OF YOUR STORIES TO
CHANGING SCIENCE.
FIRST WE'LL START OFF WITH A
GROUP OF INCREDIBLE ADVOCATES
THAT KNOW FULL WELL THAT DATA
DRIVES THE SCIENCE, THAT THE
STORIES ARE WHAT OFTEN MOVE
PEOPLE TO ACT.
THEY HUMANIZE THE COMPLEX
SCIENCE, BUILD COMMUNITY.
WE USE IT IN FUNDRAISING.
BUT THEY BRING URGENCY AND
CLARITY TO RESEARCHERS,
POLICYMAKERS, THEY ARE OFTEN
MORE IMPORTANT SOMETIMES THAN A
WHITE PAPER OR PUBLICATION.
WE'LL EXPLORE ALL OF OUR STORIES
TODAY BUT WE'RE ALSO GOING TO
ASK YOU TO THINK THROUGH THE
PRACTICAL INSPIRATION AND THE
PRACTICAL INSPIRATION THAT
STORIES BRING BUT THEN THE POWER
OF THAT TO CHANGE SYSTEMS FOR
THE PEOPLE THAT ARE LIVING WITH
THESE CONDITIONS.
I WANT TO START BY SAYING THAT I
AM THE VICE PRESIDENT OF A
PATIENT ADVOCACY ORGANIZATION,
CACNA1A FOUND IDENTIFICATION, A
MOM TO A 7-YEAR-OLD DAUGHTER
WITH THE RARE CONDITION,
ATTORNEY BY BACKGROUND, TRAINED
MY ENTIRE LEGAL CAREER ABOUT
TELLING MY CLIENTS' STORIES.
I WAS NOT USED TO TELLING MY OWN
STORY, NOT COMFORTABLE SHARING
MY OWN STORY AFTER MY DAUGHTER
WAS DIAGNOSED BUT UNDERSTOOD
THAT WAS AN ESSENTIAL FEATURE OF
OUR ABILITY TO MOVE THE SCIENCE
FORWARD IN PATIENT ADVOCACY
SPACE.
OVER FIVE YEARS SINCE HER
DIAGNOSIS I HAVE REFINED AND
CONTINUED TO TELL OUR STORY IN A
NUMBER OF DIFFERENT FORUMS.
I THINK WE STARTED THIS ENTIRE
NON-PROFIT FORUM WITH THE STORY
OF REPRESENTATIVE WEXTON, AND
RON SHARING HIS STORY.
WE HAVE ACCESS TO SOCIAL MEDIA,
DIFFERENT OUTLETS, AND WE'RE NOT
FIGHTING FOR SOMEONE TO LISTEN
BECAUSE WE CAN PUT IT OUT THERE.
WE'LL EXPLORE ALL THOSE THEMES
TODAY.
FIRST, JENNIFER, SHARE MAYBE
YOUR PERSONAL STORY THAT BROUGHT
YOU INTO THE DISEASE ADVOCACY
SPACE.
>> THANK YOU.
JENNIFER SILLS FROM SAN
FRANCISCO, CALIFORNIA.
IN 2016 MY DAUGHTER WAS SIXTH IN
THE WORLD TO BE DIAGNOSED WITH
OCHER CHON SYNDROME, WE SEARCH 7
YEARS TO FIND WHY SHE HAD
DIFFICULTY WALKING, EATING,
TALKING.
OUR DOCTOR HANDED US THE ONLY
INFORMATION OUT THERE WHICH WAS
A SINGLE PUBLICATION DESCRIBING
THE SYNDROME, AND SAID GOOD
LUCK.
AND I WENT HOME, I WAS AN
ENGLISH MAJOR AT BERKELEY,
THOUGHT I WAS HOT STUFF.
I REALIZED I HAD TO LOOK UP
EVERY OTHER WORD TO UNDERSTAND
MY DAUGHTER'S DIAGNOSIS.
I KNEW WE HAD TO DO BETTER.
I LOOKED UP THE AUTHOR, DR.
WENDY CHUNG FROM COLUMBIA, AND I
CALLED HER.
I HAD TWO PAGES OF NOTES.
I WAS AN ATTORNEY, LIKE SUNITHA.
I WAS THOROUGH.
WE GOT THROUGH FOUR QUESTIONS.
SHE TOLD ME THE COLD HARD FACTS
OF RARE DISEASE, WHICH WAS NO
ONE WAS GOING TO BE STUDYING
THIS DISEASE UNLESS WE DID IT
OURSELVES.
AND SO SHE TOLD ME TO BUILD AN
ARMY, TO GO OUT AND FIND 60
OTHER FAMILIES, TO HOST AN
IN-PERSON CONFERENCE, TO MAKE A
MOUSE MODEL, AND TO FUND BASIC
RESEARCH.
AS I STARTED ON THE JOURNEY HOW
WAS I GOING TO GET RESEARCHERS
TO A CONFERENCE, TRUST FAMILIES
TO JOIN US?
STORYTELLING.
HOW WAS I GOING TO FIND MONEY
FOR BASIC RESEARCH?
STORYTELLING.
I THINK AS WE'VE SEEN TODAY
SINCE THE -- THE ACRONYMS THROW
ME
CSNK2A1, WHAT A MOUTHFUL.
THE NINDS THAT WAS STARTED BY
STORYTELLING.
A WOMAN WENT TO CONGRESS AND
TOLD HER STORY.
IT'S EASIER TODAY THAN FOR RON,
WE HAVE SOCIAL MEDIA AND CAN
LOOK LIKE PROFESSIONALS USING
CANVA, TIKTOK TO DO 60 OR
90-SECOND REELS TO GET OUR
MESSAGE OUT THERE.
STORYTELLING WILL HOPEFULLY
BRING TREATMENT TO MY DAUGHTER.
>> GOOD AFTERNOON.
I'M GARDNER LAPHAM, AND I'M
HERE, WHAT BROUGHT ME IN 17
YEARS AGO WAS AFTER I LOST MY
SON, HENRY, HE WAS 4.
HE HAD FEBRILE SEIZURES WHEN
APPROXIMATE YEARS OLD, WE WERE
TOLD NOT TO WORRY, THEY GROW OUT
OF IT.
I WAS A NURSE.
I GET IT.
TURNED 4, AFEBRILE SEIZURE, THEN
A SECOND ONE.
WE WENT TO THE NEUROLOGIST WHO
PUT HIM ON MEDICATION, WE BEGAN
HIS WORKUP FOR EPILEPSY.
AND BEFORE HE WAS THERAPEUTIC
MEDICINE, A FULL WORKUP, HE DIED
IN HIS SLEEP.
I FOUND HIM IN HIS BED ONE
MORNING.
AND, YOU KNOW, I'M STILL KIND OF
IN SHOCK THAT THIS HAPPENED.
WE WERE ALL BLIND SIDED.
WE DIDN'T KNOW A SEIZE SEIZURED
TAKE A LIFE LIKE THAT.
WE MET WITH THE NEUROLOGIST
ONCE.
I REMEMBER SAYING CAN YOU DIE
FROM A SEIZURE, HOW WORRIED
SHOULD WE BE?
AGAIN, THIS WAS 17 YEARS AGO.
THE ADVICE WAS THE SAFEST THING
IS LET YOUR CHILD SLEEP ALONE.
I COULDN'T ACCEPT THAT.
I WAS ALSO TOLD IT'S SO RARE,
DOESN'T HAPPEN.
THERE'S NOTHING YOU COULD HAVE
DONE.
IT WAS A ONE IN A ZILLION EVENT.
SO I JUST COULDN'T BELIEVE IT
DIDN'T HAPPEN TO ANYONE ELSE.
NINDS, THANK YOU.
I WAS THINKING IN THIS MEETING,
I WENT ON THE WEB, NOTHING ABOUT
WHY HENRY DIED.
FROM SOMETHING CALLED SUDDEN
UNEXPECTED DEATH IN EPILEPSY.
THERE WAS ONE ORGANIZATION IN
THE UK, A PATIENT-FACING GROUP,
BUT NOTHING IN THE U.S.
THERE WAS A RESEARCH PAPER, SAME
THING AS YOURS, A RESEARCH PAPER
OUT OF NINDS, WE'VE GOT NEW
EPILEPSY BENCHMARKS.
I FOUND A NAME, ANN BERG, I
REACHED OUT.
WITH MY STORY IN AN E-MAIL
THINKING THEY WOULD NEVER
RESPOND.
AND SHE DID AND PUT ME IN TOUCH
WITH ANOTHER PATIENT GROUP THAT
FUNDS RESEARCH IN THE EPILEPSY
SPACE, CURE EPILEPSY, THAT'S
WHAT BEGAN MY JOURNEY IN THIS
SPACE.
AND SINCE THEN I'VE GONE ON TO
WORK WITH A FEW ORGANIZATION
ALSO BUT WHAT I DO NOW IS
CO-CHAIR SOMETHING CALLED
PARTNERS AGAINST MORTALITY IN
EPILEPSY, AND I ALSO AM PART OF
A FAMILY FOUNDATION THAT FUNDS
SUDEP, MORTALITY PREVENTION IN
THE U.S., WORK ON EPILEPSY IN
AFRICA AND HOW DO WE NARROW THE
TREATMENT GAP.
THAT'S IT IN A NUTSHELL.
>> INTERESTING TO HEAR YOUR
STORIES, SO MANY OF US HAVE
SIMILAR STORIES AND WE HEAR IT
THROUGH OUR FAMILIES ALL THE
TIME.
YOU KNOW, OUR STORIES TOES SEEM
ISOLATING, BUT THERE'S
SIMILARITIES.
AFTER MY DAUGHTER'S DIAGNOSIS I
WAS NOT GIVEN ANY EVEN -- NOT
EVEN A PAPER.
AND WAS TOLD THERE'S NOTHING
THAT COULD BE DONE, AND TO GO
HOME AND LOVE MY CHILD AND SAME
THING THAT A LOT OF PEOPLE HEAR.
WE FOUND A PAPER, COLD E-MAILED
CLINICIANS AND RESEARCHERS.
BECAUSE OF THE STORY, EVEN IN
THE SUBJECT LINE, MY DAUGHTER
WAS JUST DIAGNOSED WITH THIS, WE
NEED HELP, WE GOT RESEARCHERS
AND CLINICIANS TO RESPOND TO
THESE COLD E-MAILS WHICH I'M SO
GRATEFUL FOR IT TO THIS DAY.
BUT I JUST WANT TO SORT OF
DOUBLE BACK ON WE UNDERSTAND THE
IMPORTANCE OF STORYTELLING BUT
WHAT HAS THAT MADE POSSIBLE IN
YOUR JOURNEYS AS PARENTS AND
ADVOCATES, FOR THE MISSIONS OF
THE ORGANIZATIONS YOU'RE
INVOLVED WITH?
>> FROM THE BEGINNING I SAID IT
BROUGHT EVERYTHING TO BE US, TO
BE HONEST, FROM ENGAGING
RESEARCHERS, BUILDING THIS
NETWORK OF 45 RESEARCHERS NOW
AROUND THE WORLD, HAVING DONOR
SUPPORT.
THEN EMPOWERING FAMILIES TO TELL
THEIR OWN STORY.
I FEEL LIKE WHEN FAMILIES KNOW
HOW TO TELL THEIR OWN STORY THEY
ARE BETTER ADVOCATES AT THE
DOCTOR'S OFFICE, WHEN FIGHTING
FOR IEP RIGHTS, SO I BELIEVE
THAT STORYTELLING IS WEAVED INTO
EVERY FACET OF THIS JOURNEY.
>> I TOTALLY AGREE.
I DIDN'T KNOW GETTING INTO THIS
THAT STORYTELLING WAS GOING TO
BE SO INTEGRATED INTO EVERYTHING
WE DO.
BUT WITH THE GROUP, WITH
CLINICIANS, RESEARCHES, OTHER
FAMILIES, ADVOCATES, WE FORMED
PARTNERS AGAINST MORE PALLET IN
EPILEPSY, WHICH IS A
COLLABORATIVE, HOUSED WITHIN THE
AMERICAN EPILEPSY SOCIETY.
BUT REALLY THE CORNERSTONE IS
BRINGING THE FAMILY STORIES.
WE HAVE A CONFERENCE EVERY YEAR,
EVERY SESSION STARTS WITH A
STORY.
WE HAVE WEBINARS, THEY ARE PART
OF EVERYTHING WE DO.
AND WHEN I FIRST GOT INTO THIS,
NO ONE TELLS YOU HOW TO DO THIS
THERE'S NO ROAD MAP, IT'S
LONELY.
I FELT MY WAY AS I WENT.
YOU KNOW IT'S IMPORTANT AND IT'S
THE ONLY WAY, THE BRINGS YOU TO
THE TABLE, TO BE ABLE TO TELL
YOUR STORY, GIVES YOU
LEGITIMACY.
BUT IT REALLY NOT ONLY DOES IT
HELP MOVE ISSUES BUT HELPS
CONNECT WITH OTHER FAMILIES AND
BRING THEM INTO MORE TOO.
AS YOU SAID, EVERYONE IS SO
ALONE OUT THERE.
AND THEN YOU MORE YOU GET INTO
IT, YOU REALIZE OH MY GOD, SO
MANY PEOPLE ARE OUT THERE AND WE
CAN DO SO MUCH MORE TOGETHER.
>> I'LL NEVER FORGET THE FIRST
CONFERENCE FOR THE CACNA1A
FOUNDATION, BROUGHT TOGETHER
RESEARCHERS, OF COURSE OUR
FAMILIES, A BASIC RESEARCHER
CAME UP AFTER THE FIRST DAY AND
SAID, I'VE BEEN STUDYING THIS
DISEASE FOR 20 YEARS, I'VE NEVER
MET A SINGLE PATIENT WITH THIS
DISORDER.
AND HE WAS BLOWN AWAY BY SEEING
THE LIVED EXPERIENCE, SEEING HOW
THESE KIDS WERE IMPACTED, HOW
PARENTS AND CAREGIVERS HAD TO
RESTRUCTURE THEIR LIVES AFTER
THESE DIAGNOSES.
HE WAS GOING BACK TO THE LAB
WITH A DIFFERENT SENSE OF
PURPOSE AND DIFFERENT MISSION IN
MIND ALMOST.
I THINK THAT WAS ONE JUST REALLY
POWERFUL EXAMPLE OF BRINGING
BASIC RESEARCHER WHO MAY NOT
HAVE THE PATIENT IN MIND
THROUGHOUT THEIR CAREER TO THE
TABLE, AND KIND OF EMBEDDED IN
OUR MISSION.
CAN YOU TALK ABOUT HOW YOU
SHIFTED YOUR STORIES TO ADVANCE
YOUR MISSION, WHETHER
FUNDRAISING, RESEARCHES, OTHER
FAMILIES, HOW DO YOU THINK ABOUT
TAILORING YOUR STORY FOR
DIFFERENT PURPOSES AND
AUDIENCES?
>> YEAH, SOMETIMES WE MAY NOT
THINK ABOUT STORYTELLING, IT
TAKES TIME, MONEY IF YOU WANT TO
HIRE SOMEONE TO HELP YOU CRAFT
YOUR STORY.
SO WHEN I THINK ABOUT THE EVERY
LIFE FOUNDATION WHEN GOING TO
D.C., THE CLOSEST I EVER GOT TO
ADVOCATING WAS SLEEPING ON MY
FRIEND'S COUCH IN THE SUMMER
WHEN THEY WORKED ON THE HILL SO
THEY HELPED ME THINK ABOUT
CRAFTING THE STORY, HOW DO I GO
AND TALK TO A STAFFER AND RELATE
TO ISSUES.
BIG ON OUR MINDS, MY COMMUNITY,
IS TELEHEALTH.
NUMBER ONE EXPERT IN THE COUNTRY
IS NO NEW YORK.
SO THEY CAN'T SEE HER.
SO TELEHEALTH WOULD BE VALUABLE
TO THEM.
AND FOR MANY OTHER REASONS AS
WELL.
HOW IS IT THAT WE ARE ABLE TO
CONVEY THIS MESSAGE, A BILL IS
COMING ACROSS YOUR DESK, VOTE
FOR TELEHEALTH, CHECK THE BOX.
THINKING ABOUT WHAT VISUAL WE
COULD BRING ALONG, AND HOW WE
COULD RELATE TO THEIR OWN STORY.
I REMEMBER TALKING ABOUT IMAGINE
IF YOU WERE LIVING IN A STATE
AND YOU MOVED, EVEN IF IT WAS
JUST FOR COLLEGE.
YOU COULDN'T SEE -- YOU WERE
SEEING A PSYCHIATRIST OR
THERAPIST, YOU INVESTED IN
FINDING THE RIGHT PERSON AND YOU
JUST GO TO COLLEGE TO ANOTHER
STATE, DUE TO THE RULES, YOU
CAN'T SEE THAT PERSON ANYMORE
BUT OVER TELEHEALTH YOU COULD
STILL POSSIBLY SEE THAT PERSON.
I RELATED THAT BACK TO THE RARE
DISEASE STORY ABOUT THEIR BEING
ONE EXPERT IN THE COUNTRY AND
WHAT THAT DOES TO PATIENT
POPULATION.
I WOULD SAY MOST, 99.9% GOT IT
AT THAT POINT, WHEN I JUST
TALKED ABOUT RARE DISEASE IT
GLAZES RIGHT OVER.
MOST PEOPLE CAN UNDERSTAND THE
MENTAL HEALTH ASPECT SO I WAS
ABLE TO INTERTWINE THOSE.
I DO THINK AS FAR AS WHEN
FOUNDATIONS ARE THINKING ABOUT
STRATEGY, ABOUT MOVING THE
NEEDLE, STORYTELLING IS A REALLY
IMPORTANT INVESTMENT IN THAT
PROCESS.
>> YEAH, WE KNOW WE HAVE TO
CONNECT OUR STORIES TO THE
AUDIENCE AND UNDERSTAND WHAT
THEIR PRIORITIES ARE, TRY TO
MEET THEM THERE.
SO IN SUDEP, I'VE MET SO MANY
BEREAVED FAMILIES ALONG THE WAY.
SOME THING THAT RINGS TRUE, WHAT
MOST PEOPLE SAY, I DIDN'T KNOW
IT COULD HAPPEN, MY DOCTOR
DIDN'T TELL ME MY KID COULD DIE
AND MAYBE SOMETHING WE COULD
HAVE DONE.
I GET IT, WHEN MY SON DIED NO
ONE WAS TALKING ABOUT IT.
LIKE NOW EVERYBODY SHOULD BE
TALKING ABOUT IT.
IT'S IN THE GUIDELINES.
YOU HAVE TO PICK THESE THINGS
OFF ONE AT A TIME.
SO THE PERSONAL STORIES AND
RELATIONSHIPS, LIKE A STORY
DEVELOPS A RELATIONSHIP, AND
THAT HAS BEEN MEANINGFUL TO SEE
PRACTICE CHANGE.
ADVOCATES I WORK WITH, SPEAKING
WITH CLINICIANS, TALKING TO
PEOPLE ABOUT HOW TO -- OFTEN
PEOPLE DON'T TALK ABOUT █THIS
TOPIC BECAUSE THEY DON'T KNOW
HOW.
IT'S HARD.
AND SO PERSONAL STORIES CAN
INSPIRE INDIVIDUAL BEHAVIOR
CHANGE AND ACTION.
WHAT'S HARDER IS SYSTEM CHANGE,
RIGHT?
SO FINDING THE RIGHT PARTNERS TO
MARRY THE STORIES WITH THE OTHER
PIECES TO MAKING CHANGE, FOR
EXAMPLE.
>> ONE OF THE THINGS THAT WE'VE
DONE AT THE CACNA1A FOUNDATION
IS SET UP AN OPPORTUNITY ON THE
WEB PAGE FOR FOR PEOPLE TO TELL
THEIR STORIES.
EVERY MONTH WE GET AN E-MAIL
FROM A NEWLY DIAGNOSED FAMILY
SAYING THAT WAS THE MOST
IMPORTANT RESOURCE WE HAD AFTER
OUR DIAGNOSIS BECAUSE THEIR
CLINICIAN OFTEN CAN'T TELL THEM
ANYTHING ABOUT WHAT OTHER KIDS
LOOK LIKE, WHAT OTHER PATIENTS
LOOK LIKE WITH THIS DISEASE.
AND SO THEY WILL LITERALLY READ
THE 20-PLUS STORIES WE PUT UP
THERE AND WE CONSTANTLY
ENCOURAGE FAMILIES TO SHARE
THEIR STORIES WITH US BECAUSE
IT'S SUCH A SOURCE OF HOPE AND
COMFORT TO HEAR FROM OTHER
FAMILIES.
ONE OF THE THINGS, JENNIFER,
YOU'VE BEEN WORKING ON, YOU'VE
ALREADY HAD, GETTING FAMILIES
COMFORTABLE TELLING THEIR
STORIES IN FRONT OF REGULATORS
AND POLICYMAKERS, NOT JUST YOU
AS THE LEADER OF YOUR
ORGANIZATION BUT REALLY
EMPOWERING YOUR COMMUNITY.
CAN YOU TALK ABOUT THAT JOURNEY
AND GETTING FAMILIES TO OPEN UP
AND FEEL COMFORTABLE GOING
BEFORE POTENTIAL CHANGE-MAKING
ADVOCACY BODIES AND STAKEHOLDERS
TO ADVANCE THE MISSION OF YOUR
ORGANIZATION?
>> YEAH, WE ACTUALLY TRY TO
PRACTICE STORYTELLING ACROSS
STAKEHOLDERS, SO WE -- WHETHER
THAT'S WHEN GOING INTO AN IEP
MEETING, GIVING A POWERPOINT
PRESENTATION, HELPING THEM
NAVIGATE HOW TO TALK ABOUT THE
SYNDROME, AT YOU ARE CONFERENCE
THIS SUMMER SOME PEOPLE MAY KNOW
POD CASTER, EFFIE PARKS, ONCE
UPON A GENE, COMING TO RUN A
WORK
WORKSHOP TALKING ABOUT
STORYTELLING.
THE MORE -- I SAID THIS BEFORE,
THE MORE THEY CAN TALK ABOUT IT
AMONGST THEMSELVES, I THINK JUST
EMPOWERS THEM IN SO MANY OTHER
WAYS TO MAKE A DIFFERENCE.
AVENUES TO MAKE AN INVESTMENT.
>> ARE THERE SPECIFIC TRAININGS
OR TOOLS THAT YOU FOUND OUT
THERE THAT HAVE BEEN HELPFUL FOR
CRAFTING STORIES?
>> NO!
I MEAN, TO BE HONEST, I HAVEN'T
REALLY LOOKED.
IT WAS PREPARING FOR THIS, WOW,
WE NEED TO DO ANY A MORE
INFORMED WAY.
EVERYONE KIND OF FUMBLES.
WE'RE ALWAYS NERVOUS AROUND HOW
DO YOU MARRY THE STWOAR A
MESSAGE, RIGHT?
SO WE WALK -- THE PEOPLE WHO
PRESENT AT OUR MEETINGS, WE WALK
THROUGH HOW YOU BALANCE THAT.
I THINK WE COULD DO A A BETTER
JOB.
THE POINT ABOUT SOCIAL MEDIA, WE
HAVE A FACEBOOK PAGE.
EVERYBODY DOES.
I GET DATA EVERY MONTH FROM THAT
THING.
LIKE THE PERSONAL STORIES ARE --
I MEAN, OFF THE CHARTS, HIGH
CLICK-THROUGHS, WHATEVER YOU
CALL IT.
I'M ALWAYS AMAZED.
GOSH, WE DON'T HARNESS THIS AS
MUCH AS WE SHOULD OR WE COULD.
BECAUSE IT IS HARD.
WE DO A REMEMBRANCE WALL, AND
IT'S LIKE PEOPLE WANT TO SHARE
THEIR STORIES, AND PEOPLE ARE
WILLING TO PUT THEIR LOVED ONES
UP THERE.
IT'S ALWAYS AMAZING TO ME SO
MANY PEOPLE THAT WANT TO SHARE.
I'M ALWAYS STRUGGLING, HOW DO WE
DO THAT?
IT'S A HUGE RESPONSIBILITY.
BUT I THINK WHAT YOU'RE DOING,
HOW TO TELL THEIR STORIES,
THAT'S A GREAT IDEA.
IT IS A PROFESSIONAL THING TO
DO.
YOU WERE TALKING ABOUT THE MOTH
EARLIER, I THINK ABOUT TED
TALKS, WE NEED MORE OF THOSE
PEOPLE IN OUR SPACE TO HELP US
PROFESSIONALIZE HOW WE COMMUNAL
INDICATE.
TO INSPIRE PEOPLE TO KNOW
STORIES ARE VALID AND IMPORTANT
BECAUSE WE CAN BE INTIMIDATED BY
THIS WORLD, YOU KNOW, IF YOU'RE
NOT FROM THE MEDICAL WORLD, TO
KNOW YOUR VOICE MATTERS, THAT
THERE'S EQUAL PARTNERSHIP MAKING
THIS STUFF ADVANCE.
>> ONE OF THE THINGS I'M GOING
ASK YOU TO TALK ABOUT, WHEN WE
GET TO THE AUDIENCE Q&A, THE
STORY COLLIDER PROGRAM AND
TRAINING THAT THE CHAN
ZUCKERBERG INITIATIVE GAVE
ACCESS TO GRANTEES WAS AROUND
STORYTELLING, BUT THE OTHER
OPPORTUNITY I WAS TALKING ABOUT
EARLIER WITH PANELISTS, AT BIO
LAST YEAR, PATIENT ADVOCACY
SUMMIT THEY BROUGHT IN TRAINERS
TO HELP ADVOCATES CRAFT AND
REFINE THEIR STORY AND GET
TRAINED HOW TO DELIVER THE
STORY.
OFTENTIMES EXECUTIVES GET THIS
TRAINING AND THEY INVEST IN THAT
TRAINING, THEY HIRE FOLKS TO
HELP THEM WITH DELIVERING
SPEECHES AND IN PUBLIC SPEAKING.
ADVOCATES ARE THRUST ON THE
SCENE FROM THE START WITHOUT ANY
OF THAT.
SO I THINK INVESTING IN THAT IS
REALLY IMPORTANT.
EILEEN PENMILLER SUGGESTED
ORGANIZING A WORKSHOP IN THE
RARE EPILEPSY SPACE.
WE'LL FOLLOW UP WITH YOU,
EILEEN, ON THAT.
GO AHEAD, JENNIFER.
>> THE OTHER THING, IT DOESN'T
HAVE TO COST A LOT OF MONEY
EITHER, IS THAT WE HAD AN INTERN
THAT CAME FROM THE PUBLIC POLICY
SCHOOL AT UC-BERKELEY, I RAN OUT
OF IDEAS OF WHAT TO HAVE HER DO
FOR US.
I WENT TO ChatGPT AND GAVE IT
A PROMPT, COME UP WITH TEN
PROJECTS.
THEY WILL BRILLIANT, ONE WAS TO
INTERVIEW FAMILIES, CHOOSE TEN
TOPICS RELEVANT TO THE
COMMUNITY, HAVE THEM DO
15-MINUTE INTERVIEWS, WRITE A
3-PARAGRAPH ESSAY OR BLOG, NOT
ESSAY, BIT. -- ABOUT IT.
WHY WOULDN'T I ChatGPT MORE?
WE PLUGGED IN AND SAID WRITE A
60 TO 90 MINUTE SCRIPT FED BY
THE FAMILY WHO DID THE INTERVIEW
THAT THEY COULD DO AN AUDIO
RECORDING OF AND PROVIDE US WITH
VIDEOS AND PICTURES THAT WE
COULD USE AS B ROLL WITH THEIR
VOICEOVER TALKING ABOUT WHATEVER
THE TOPIC WAS RELATED TO THEIR
EXPERIENCE.
AND IT WAS AMAZING.
FIRST PEOPLE WANTED TO TELL
THEIR STORIES.
TWO, OTHER PEOPLE FEEL SEEN WHEN
READING THE STORY IF THEY HAD A
SHARED EXPERIENCE.
AND WE WERE ABLE TO USE AN
ASSET, THIS THREE-PARAGRAPH
ASSAY, ACROSS A COUPLE
MODALITIES, DOING FILMS, NOT
REALLY FILMS, A REEL, AND THEN
ASKED ChatGPT TO PULL OUT A
QUOTE FROM THE INTERVIEW AS WELL
SO WE COULD USE THAT AS A TITLE
THAT OUR FAMILIES COULD RELATE
TO.
STORYTELLING WHEN I THINK ABOUT,
I GO BACK TO RON, 27 YEARS AGO,
HOW CRAFTY YOU HAD TO BE TO TELL
YOUR STORY AND WE HAVE SO MANY
WAYS TO TELL IT NOW AND REUSE
IT.
DON'T BE AFRAID TO USE A.I.,
WORK SMARTER NOT HARDER, AND
JUST THINK OF DIFFERENT WAYS
THAT YOU CAN -- IT DOESN'T HAVE
TO BE THREE PARAGRAPHS LONG,
COULD BE ONE QUOTE THAT'S REALLY
POWERFUL THAT STILL TELLS THE
STORY.
>> NO ONE CAN READ THREE
PARAGRAPHS ANYMORE.
[LAUGHTER]
OTHER FORMATS OR MEDIUMS YOU USE
THAT HAVE BEEN EFFECTIVE OR --
>> I LOVE YOUR IDEAS.
YOU'VE GIVEN ME AN IDEA FOR MY
19-YEAR-OLD.
ON THE OTHER SIDE OF THE
SPECTRUM WHEN IT COMES TO -- OH
RESOURCES, MORE RESOURCE
INTENSIVE THING TO DO IS A FILM
THAT I HAD THE OPPORTUNITY TO
CO-PRODUCE WITH ANOTHER
FOUNDATION AND WE RECOGNIZED
THERE'S SUCH AN ENORMOUS PROBLEM
OF EPILEPSY AROUND THE WORLD,
ESPECIALLY LOW-INCOME COUNTRIES,
MORE THAN 80% OF PEOPLE DON'T
RECEIVE ANY TREATMENT AT ALL.
HERE WE ARE IN THIS
WELL-RESOURCED COUNTRY, NO ONE
KNOWS ABOUT THIS.
REALLY, NO ONE TALKS ABOUT
GLOBAL EPILEPSY.
WE DECIDED TO TRY AND TELL THE
STORY AND SHINE A LIGHT ON THE
ISSUE.
WE WORKED WITH A FILM PRODUCTION
COMPANY HERE, WENT TO KENYA,
INTERVIEWED FOUR FAMILY STORIES,
AND CREATED A 17-MINUTE
DOCUMENTARY.
IT WENT TO 20-PLUS FILM
FESTIVALS.
WE'VE TAKEN IT ON THE ROAD AND
SHOWED IT AT ALL KINDS OF
SETTINGS REALLY, LIKE ACADEMIC
SETTINGS, NOW HOSPITALS, SCHOOLS
OF PUBLIC HEALTH.
BUT WE'VE ALSO TAKEN IT TO DONOR
NETWORKS AND HAD PANEL
CONVERSATIONS.
WE'VE LEARNED THIS IS AN ENTRY
POINT FOR PEOPLE TO TELL THEIR
STORIES.
IT'S LIKE WATCH THIS SHORT
DOCUMENTARY AND LEARN ABOUT
SOMETHING YOU DON'T KNOW MUCH
ABOUT, AND YOU CAN TALK ABOUT
EPILEPSY HERE AND ABROAD BUT THE
BIG THEME IS HOW UNDERRESOURCED,
HOW URGENT THE PROBLEM IS.
WE CREATED A CAMPAIGN THAT RUNS
ALONGSIDE THE FILM TO PROVIDE A
WAY FOR PEOPLE TO SUPPORT PEOPLE
ON THE GROUND IN AFRICA .
AS PART OF THIS WORK WE'RE
HELPING ENABLE ADVOCATES TO TELL
THEIR STORY, CHANGE POLICY IN
THEIR OWN PARTNER ORGANIZATION,
AND DEVELOPED AN ADVOCATE TOOL
KIT, PART IS STORYTELLING.
THERE'S OTHER STUFF.
NOW I'M THINK WE GO SHOULD DO A
STORYTELLING TOOL KIT BECAUSE
THERE'S SO MUCH, YOU'RE RIGHT,
TO DO IT REALLY WELL.
I THINK WE TAKE IT FOR GRANTED,
YOU KNOW, BUT IT'S HARD.
IT'S A THE CONGRESSWOMAN TODAY
ABOUT A PHENOMENAL JOB.
>> ONE OF THE THINGS DO, WE'VE
HAD TWO CONFERENCES BUT ALWAYS
HAVE A FILMMAKER, LOW BUDGET,
COULD BE A STUDENT WHERE YOU'RE
HAVING YOUR CONFERENCE BUT HAVE
TIME FOR FAMILIES TO BE
INTERVIEWED AT OUR CONFERENCE,
AND RESEARCHERS AND CLINICIANS,
TELL THE STORY HOW WE WORKING
TOGETHER HAS BEEN HELPFUL NOT
ONLY TO RAISE AWARENESS BUT FOR
FUNDRAISING PURPOSES, WE USE IT
WITH THE END OF YEAR CAMPAIGN,
SPONSORS, SUPPORTING EVENTS LIKE
THIS.
THERE'S SO MANY MEDIUMS TO
UTILIZE YOUR STORY BEYOND
RAISING AWARENESS.
I WANT TO TOUCH ON HOW TO TELL
YOUR STORY AND BALANCE BEING
EMOTIONALLY OPEN AND VULNERABLE
WITH ADVOCATING FOR A REAL
PURPOSE.
LIKE THERE'S SO MANY STORIES
THAT ARE HEART WRENCHING IN SO
MANY WAYS BUT HOW DO YOU ALSO
WEAVE IN LIKE WHAT YOU'RE TRYING
TO GET OUT OF THE STORYTELLING,
WHY YOU'RE TELLING THE STORE IN
THE FIRST PLACE, THE BALANCE
BETWEEN STRATEGY AND
VULNERABILITY.
>> YOU HAVE TO REMEMBER IT
DOESN'T HAVE TO BE TRAGIC TO BE
EFFECTIVE.
THERE'S MANY STORIES THAT WE
HAVE TO TELL THAT RELATE TO RARE
DISEASE.
I THINK THE MORE VULNERABLE THAT
WE ARE, THE DEEPER WE CONNECT
WITH OTHER PEOPLE.
IF YOU'RE MY FRIEND ON FACEBOOK,
I SOMETIMES LIKE TO WRITE,
REMEMBER I WAS AN ENGLISH MAJOR,
AND I WILL WRITE SOMETHING AND
SOMETIMES WILL SIT ON IT FOR A
YEAR BEFORE I'LL PUBLISH IT.
ONE, MAYBE IT'S TOO RAW FOR ME
TO EVEN PUT IT OUT THERE BECAUSE
I, YOU KNOW, SOMETIMES PEOPLE
THINK THAT YOU'RE TRYING ELICIT
COMFORT, WHICH I'M NOT.
I JUST WANT TO TELL THE STORY.
I WILL WAIT A YEAR.
SO THAT WAS -- OH, I READ YOUR
POST, THAT WAS A YEAR AGO.
WE'RE GOOD.
YEAH, I THINK -- I KNOW THERE'S
LIKE THE PRIVACY ASPECT OF OUR
OWN CHILDREN AND THINGS LIKE
THAT SO I TRY TO TALK ABOUT MY
EXPERIENCE AND NOT HERS, BECAUSE
SHE DOESN'T HAVE A VOICE.
SHE IS 16 NOW.
I'M PRETTY CERTAIN SHE WOULDN'T
WANT ME TO TELL SOME OF THE
STORIES SO I TELL STORIES HOW I
AND MY PERSPECTIVE AND I THINK
CAN SHED ENOUGH LIGHT ON OUR
LIVED EXPERIENCE
>> I THINK IT'S A GOOD QUESTION.
TRICKY BECAUSE YOU HAVE TO
BALANCE EMOTION WITH INSIGHT,
AND THE EMOTION GRABS PEOPLE'S
ATTENTION, BUT THE INSIGHT
ALLOWS FOR CHANGE AND YOU HAVE
TO BE CLEAR.
WHO ARE YOU TALKING TO?
WHAT DO YOU WANT OUT OF IT?
I DON'T NEED TO SAY IT LIKE THAT
BUT YOU KNOW WHAT I MEAN.
SO I THINK YOU'RE RIGHT ABOUT
BEING VULNERABLE.
WE HAVE VERY DIFFERENT
EXPERIENCES BECAUSE MY CHILD'S
NOT ALIVE AND I STILL LIKE HATE
GOING TO THE COCKTAIL PARTY AND
TALKING ABOUT MY KIDS BECAUSE
YOU'RE LIKE DEBBIE DOWNER.
IT'S REALLY HARD TO GO INTO
THOSE SETTINGS, NOT THE COCKTAIL
PARTY BUT LIKE, YOU KNOW,
BECAUSE YOU HAVE TO PROVIDE HOPE
AND A WAY THROUGH.
YOU HAVE TO SAY WORK WITH ME
HERE.
HERE IS THIS TERRIBLE TRAGIC
STORY, BUT WE CAN WORK TOGETHER
TO MAKE IT BETTER FOR OTHER
PEOPLE.
AND SO I PROBABLY TO A FAULT TRY
TO FOCUS MORE ON THAT.
HOW TO TELL A STORY THAT HAS A
BAD ENDING.
>> I'M GOING TO OPEN UP TO THE
AUDIENCE ENGAGEMENT.
I WANT TO HEAR OTHER PEOPLE'S
STORIES BUT TIPS AND LESSONS
LEARNED FROM YOUR JOURNEYS.
BEFORE THAT WOULD LOVE TO HEAR
WHAT DO YOU WISH YOU HAD KNOWN
ABOUT STORYTELLING, WHEN YOU GOT
INTO THIS SPACE OR ANY ADVICE ON
INTEGRATING STORYTELLING, INTO
YOUR ORGANIZATION OR ADVOCACY
WORK?
>> I SHOULD HAVE KNOWN THAT
QUESTION WAS COMING.
WELL, TO BE HONEST WHAT I DIDN'T
EXPECT FROM IT AND I KNOW I KEEP
ON LOOKING AT YOU, RON, YOU'VE
BEEN DOING THAT I WHILE, IS THAT
I STARTED IN 2016.
WE DIDN'T LAUNCH OUR FOUNDATION
UNTIL 2018 BECAUSE IT DIDN'T
HAVE TO BE RIGHT BUT I JUST
DIDN'T WANT TO LAUNCH WITHOUT
HAVING A PLAN.
BUT RECENTLY I'VE FELT
STORYTELLING FATIGUE.
I DON'T WANT TO TELL THE STORY
ANYMORE.
I DON'T KNOW IF ANYONE HAS
EXPERIENCED THAT BEFORE.
HOW YOU LIKE OBVIOUSLY WE STILL
HAVE TO DO IT, WE'VE GOT A
MEETING, WE'RE GOING TO DO IT,
BUT HOW YOU -- AND THEN
STORYTELLING FATIGUE AND
SOMETIMES WE GET USED TO TELLING
THE SAME STORY OVER TIME THAT WE
FORGET TO TELL THE NEW STORY.
THAT THERE ARE ALWAYS NEW
STORIES TO TELL AND HOW TO MAKE
SURE WE'RE WEAVING THOSE IN.
MAYBE THAT'S HOW WE PREVENT THE
FATIGUE IS BEING INVESTING EVEN
MORE TIME IN THE STORYTELLING,
I'M NOT SURE.
I DON'T KNOW, RON, IF YOU HAVE
WORDS OF ADVICE.
I'M NOT SURE IF YOU WERE -- YES.
>> AMANDA, HYDROCEPHALUS
ASSOCIATION.
STORIES BUILD TRUST.
THEY DO CONNECT.
BUT IT ALSO TAKES A LITTLE BIT
FROM YOU TOO.
WHEN YOU'RE WORKING EVERY DAY,
IN THE CONDITION MANY OF US,
YOU'RE LIVING THE EXPERIENCE OF
HYPERMEDICAL VIGILANCE OR
ADVOCATING CONSTANTLY, MY
DAUGHTER IS 24, STILL SUPPORTING
AND MANAGING AND SCAFFOLDING TO
HELP HER BE INDEPENDENT, TELLING
THE STORY AND GOING HOME AND
LIVING IT, IT TAKES PIECES OF
YOU AND IT'S EXHAUSTING,
SOMETIMES YOU JUST WANT TO STOP.
THE OTHER THING THAT WE FIND, I
HAVE TWO WOMEN ON STAFF WHO LIVE
WITH THE CONDITION, THEY ARE THE
SUPPORT AND EDUCATION TEAM.
WE'RE DEVELOPING THE RESOURCES
FROM LISTENING TO THE COMMUNITY
AND TAKING IN THE STORIES.
THOSE TWO WOMEN ARE SHARING
STORIES TO HELP PEOPLE GET
THROUGH A MOMENT IN THE HOSPITAL
OR A LONG DAY THAT DIDN'T GO
WELL FOR WHATEVER REASON.
WE WORK ON TRYING TO FIGURE OUT
HOW TO FIND SPACE FOR EACH OTHER
TO VENT OR RELEASE BECAUSE WE
TAKE HOME EVERY UPON, TWO OR
THREE MONTHS LATER CHECK ON
PEOPLE, SUPPORTING THE ENTIRE
U.S. AND ABROAD.
THESE STORIES NEVER LEEIVE OUR
MINDS.
WE LIVING AND RELIVING STORIES
AND TELL THEM.
THE OTHER THING, MY DAUGHTER IS
NOW 24.
PEOPLE ARE STILL CONNECTED TO ME
BECAUSE I'M A MOTHER AND TELL MY
STORY, BUT IT'S NOT MY STORY
ANYMORE.
MY DAUGHTER HAS BEEN OPEN,
SAYING I'M TIRED OF BEING THE
POSTER CHILD FOR HYDROCEPHALUS.
I REALLY HAD TO NAVIGATE THAT AS
PART OF OUR TRANSITION PROCESS.
ERIC AND I WERE TALKING ABOUT
TRANSITION, WE HAVE KIDS IN THE
SAME AGE RANGE.
MOVING AWAY FROM BEING ABLE TO
WALK INTO A PARTY AND YOU'RE
STANDING WITH YOUR ADULT CHILD,
OH, AND, OH, YEAH, MY DAUGHTER
HAS HYDROCEPHALUS, THAT'S NOT
FAIR.
I WORK WITH A LOT OF PARENTS IN
THE TRANSITION PHASE, SPEAKING
AT WEBINARS, CONFERENCES,
WORKING WITH OUR FAMILIES, PART
OF THE THING WE TALK ABOUT IS
WHEN DOES THE STORY NOT BECOME
YOURS ANYMORE, HOW DO YOU TELL
YOUR PIECE OF THE STORY
WITHOUT -- YOU KNOW, TAKING
CONTROL OF YOUR CHILD'S STORY
NOW THAT THEY ARE ADULTS, ON
CAPITOL HILL, ANOTHER MEDICAL
CONFERENCE, OR TO ANOTHER FAMILY
THAT NEEDS TO CONNECT.
THAT'S REALLY IMPORTANT IN A
TOOLKIT, AT WHAT POINT DOES THE
STORY NEED TO TRANSFER, TO THE
PERSON WHO OWNS IT, WHO IS THE
LIVED EXPERIENCE AND HOW DO YOU
SEPARATE YOUR EXPERIENCE AS A
CAREGIVER FROM THE EXPERIENCE
EVEN THOUGH WE'RE IN EVERY
MEDICAL APPOINTMENT, WE'RE STILL
MAKING DOCTOR APPOINTMENTS,
WE'RE AT EVERY MRI, EVERY BLOOD
DRAW, WE FEEL LIKE WE HAVE THE
CONDITION THAT OUR CHILD HAS.
IT REALLY DOES FEEL THAT WAY.
WE DON'T THOUGH.
WHEN THEY ARE OLDER THEY HAVE
THAT STORY AND WE DON'T GET TO
TELL IT WITHOUT THEIR
PERMISSION.
THAT HAS TO BE AN INTEGRAL PART
OF THE TOOLKIT.
ONE OTHER THING, TO CONNECT THE
STORYTELLING, THERE'S BEEN
CONVERSATION THROUGHOUT THE DAY
HOW DO WE MOTIVATE YOUNG
RESEARCHERS TO STAY IN OUR
SPACE.
AND THAT CONNECTION AND STORIES,
ONE THING THAT'S REALLY
IMPORTANT, MANY OF US DO IT HERE
AT NON-PROFITS, WE HAVE PATIENT
CONFERENCES, MANY DO INVITE
YOUNG SCIENTISTS AND THEY SEE
THE VARIETY AND SPECTRUM OF
INDIVIDUALS LIVING WITH THE
CONDITION.
SO MANY SCIENTISTS SAY LIKE THAT
WAS THE MOST MEANINGFUL
EXPERIENCE FOR ME.
I UNDERSTAND SO MUCH MORE NOW.
THAT'S ALSO WITH THE NEURONURSES
AND PHYSICIAN PHYSICIAN ASSISTS
CRITICAL, SO WE HAVE TRAVEL
AWARDS TO BRING SCIENTISTS TO
CONFERENCES TO SHARE POSTERS AND
TALK TO FAMILIES AND LEARN.
BUT THEY DON'T HAVE MONEY TO
COME TO CONFERENCES, SO WE ALL
NEED TO FIGURE OUT AS A
COLLECTIVE IF WE REALLY WANT
YOUNG SCIENTISTS TO STAY
INVOLVED AND WE THINK THE
STORIES AND BEING IN PARTNERSHIP
IN THE SAME SPACE AS PATIENTS
WILL BE HELPFUL WE NEED TO STEP
UP AND FIGURE OUT HOW TO FUND
THEM TO COME TO PATIENT
CONFERENCES.
JUST A THOUGHT I WANTED TO BRING
IN FROM EARLIER.
BUT THANK YOU, JENNIFER AND
GARDINER AND SUNITHA, FOR
EVERYTHING YOU'RE SHARING.
>> THERE IS ONE ORGANIZATION OUT
THERE, THEY HAVE THE YOUNG
INVESTIGATOR DRAFT.
YOU CAN APPLY AS A YOUNG
INVESTIGATOR FOR CONFERENCES, WE
NEED MORE OF THOSE.
WHEN I GO TO THE THERAPIST AND
TALK ABOUT MY STORY SHE'S LIKE,
HE JUST TALK ABOUT IT SO MATTER
OF FACT, AND THAT'S THE THING.
IF I'M TALKING TO SOMEONE HOW
EFFECTIVE CAN I BE IF I'M
ADVOCATING AT AN OFFICE OR
MEETING IF I'M A PUDDLE ON THE
FLOOR?
THERE'S ALSO THE FINE LINE OF
HOW DO YOU BE EMOTIONALLY
VULNERABLE, LIKE RON, YOU DO IT
SO PERFECTLY, AND BEING
EFFECTIVE AT THE SAME TIME.
MAYBE THAT'S WHAT PART OF THE
FATIGUE IS ABOUT, I FEEL LIKE I
NEVER ACTUALLY TELL MY STORY TO
BE HONEST BECAUSE PART OF MY
STORY IS THE HEARTACHE AND THE
FACT SHE WAS -- WHEN SHE TURNED
16 THERE WERE NO FRIENDS BUT
THAT'S NOT HOW MOST 16-YEAR-OLDS
ARE SPENDING THEIR BIRTHDAY.
THERE'S ALSO THAT PART OF
STORYTELLING WHERE THERE'S THAT
FINE BALANCE WHERE YOU DON'T
ACTUALLY REALLY TRULY GET TO GO
THERE.
>> I WANT THESE GUYS TO SPEAK.
>> HE'S GOING FIRST.
>> I'D LIKE TO GIVE A DIFFERENT
PERSPECTIVE.
SO I'M IRVING WEINBERG, I RUN A
VENTURE STUDIO A MILE FROM HERE.
WE HAVE TECHNOLOGIES, WE FIGURE
OUT WHAT WE'RE GOING TO DO WITH
THEM.
WE BUILD SYSTEMS FOR UROLOGISTS,
FOR OWNERS OF THOROUGHBRED
HORSES, BREAST IMAGING.
I WAS AT A CONFERENCE YESTERDAY
ORGANIZED BY DEPARTMENT OF
DEFENSE.
THEY DO REVERSE PITCHES.
THEY SAY THIS IS WHAT WE WANT AT
THE DIU, WHAT WE WANT AT THIS
THING.
JUST WE HAVE -- WE SET ALL
NON-PROFIT FOR COLLABORATING
WITH CHILDREN'S NATIONAL
HOSPITAL, REALLY IT'S IN THE
LAST HALF HOUR I'VE SAID WE'VE
BEEN LOOKING, WHAT PROJECT
SHOULD WE DO?
YEAH, LET'S SET UP A SCREENING
FOR SUDDEN INFANT DEATH
EPILEPSY, IT'S EASY FOR US TO
DO.
A DIFFERENT VALUE.
YOU'RE USED TO THE PROFESSORS
WHO HAVE BEEN WORKING AT ONE
THING FOR THE LAST 30 YEARS.
BUT FOR A LOT OF US IN INDUSTRY,
IT'S, WELL, WHAT SHOULD WE DO
NEXT?
IT'S NICE FOR SOMEBODY TO SAY,
YEAH, THIS WOULD BE REALLY
IMPORTANT TO ME.
WELL, WHO ELSE?
MAYBE THERE'S A MARKET.
MAYBE WE SHOULD DO THAT.
THANK YOU.
>> ONE OF THE THINGS THAT I
WANTED TO POINT OUT IS YOU
TALKED ABOUT HOW IMPORTANT THE
STORIES ARE.
I THINK YOU ALSO FORGET THERE'S
A HUGE AUDIENCE OUT THERE THAT
YOU MAY NOT HAVE THOUGHT ABOUT.
EXAMPLES THAT CAME TO MIND, USED
TO BE, YOU KNOW, YEARS AGO THE
FDA NEVER WANTED TO SEE A
PATIENT.
THEY WOULD PROHIBIT YOU FROM
BRINGING A PATIENT WHEN YOU HAD
A MEETING WITH THEM.
NOW THAT HAS CHANGED.
THEY WANT TO HEAR THE PATIENT
STORY.
WHEN WE GOT APPROVAL FOR THE
FIRST THERAPY FOR DUCHENNE
MUSCULAR DYSTROPHY WE HAD 100
PATIENTS THAT SHOWED UP TO THE
ADVISORY COMMITTEE AND THEY ALL
WANTED TO TALK.
WHAT A DIFFERENCE!
THE REASON THAT DRUG GOT
APPROVED, THE PATIENTS TOLD
THEIR STORY.
THE OTHER GROUP, I HAD NEVER
THOUGHT ABOUT, BUT IT OCCURRED
TO ME WE WERE AT JP MORGAN, ONE
OF THE BIG PARTS OF RUNNING A
COMPANY YOU HAVE TO RAISE MONEY.
SO WE HAD AN APPOINTMENT WITH
ONE OF THE PREMIER VENTURE
CAPITAL FIRMS.
HE CAME IN, HE WAS TALKING TO
ME.
HE SAID THE REASON I WANT TO
TALK TO YOU IS A FRIEND OF MINE
SAID, WHO HAS DRAVET SYNDROME,
ONE OF HER CHILDREN, THE DRUG
THAT YOU'RE DEVELOPING IS
AMAZING.
HE INVESTED $40 MILLION BECAUSE
OF HIS FRIEND.
YOU HAVE TO REMEMBER THERE'S A
MUCH BROADER AUDIENCE THAN JUST
THE SCIENTISTS OR POLICY PEOPLE.
THE OTHER EXAMPLE IS PAYERS.
DUCHENNE WAS AN EXPENSIVE
THERAPY, THERE WAS RESISTANCE TO
PAY FOR IT.
THE MOTHERS WENT AND PRESENTED
VIDEO.
NO ONE TURNS DOWN A MOTHER AND
SAYS I DON'T WANT TO PAY FOR
YOUR DRUG.
IT'S IMPORTANT TO EXPAND WHERE
YOU'RE GOING.
THE STORY IS POWERFUL BUT
AFFECTS A LOT OF PEOPLE, NOT
JUST IMMEDIATE PEOPLE, A LOT OF
PEOPLE.
>> I'M UP HERE BECAUSE SHE SAID
I HAD TO BE.
I'M OFFERING A FEW RESOURCES.
WE WERE BOTH AWARDEES FOR THE
CHAN ZUCKERBERG INITIATIVES.
ONE TRAINING WE HAD ACCESS 20
WAS STORY COLLIDER.
IT'S AVAILABLE TO THE PUBLIC.
THESE ARE PROFESSIONAL
STORY-TELLING TRAINER PEOPLE.
AND YOU CAN ONLINE SIGN UP
FOR -- I JUST CHECKED TO MAKE
SURE, FOUR 90-MINUTE SESSIONS OF
STORYTELLING COACHING IN A GROUP
ZOOM FOR $400, MONEY WELL SPENT
IF YOU HAVE PATIENTS WHO YOU
THINK ARE GOING TO BEING THE
FACE OF YOUR ORGANIZATION.
MORE ECONOMICALLY, TED TALKS
HAVE A MASTER CLASS ONLINE THAT
YOU CAN TAKE FOR $20.
WE HAVE SPONSORED MULTIPLE OF
OUR PATIENTS TO GO THROUGH THAT.
IT'S VIDEOS.
SNACKABLE VIDEOS, 5 TO 10
MINUTES, THERE'S 11 OF THEM.
IT GIVES A SENSE OF WHAT THE
STRUCTURE OF A GOOD STORY TALK
LOOKS LIKE, WHAT YOU SHOULD BE
FOCUSING ON, HOW DO YOU GET
THEMATIC.
AND THE LAST THING TO MENTION
REFLECTING ON INTRODUCING
INVESTIGATORS TO PATIENT
STORIES, WE STOLE ANOTHER IDEA
FROM CGI ORGANIZATION, TO HAVE
PATIENT POSTERS AT OUR POSTER
SESSION.
OUR POSTER SESSIONS ARE
INTEGRATED, PATIENTS HAVE
POSTERS THAT TELL THEIR STORIES.
ALSO THERE'S ALWAYS A BLURB THAT
SAYS WHAT WE WANT RESEARCHERS TO
KNOW.
AND THAT ALLOWS THE RESEARCHER
TO HEAR THE PRIORITY FOR THE
PATIENT.
AND IN THE SAME SESSIONS ARE THE
RESEARCHER POSTERS SO THE
PATIENTS HAVE THE OPPORTUNITY TO
SEE THE WORK THAT'S BEING DONE.
THANKS.
>> AND I'VE GOT ONE FINAL STORY.
I'D LIKE TO PUT IN A PERSONAL
PLUG FOR BEING EMOTIONALLY
AVAILABLE.
YOU ALL DEALT WITH THAT
POSSIBILITY, SOME OF ITS
DRAWBACKS.
I WOULD SAY BEING EMOTIONALLY
AVAILABLE IS POWERFUL.
IT'S A POWERFUL PART OF YOUR
STORY.
IT'S A POWERFUL PART OF YOUR
STORY FOR YOUR OWN FAMILIES.
THEY GET EMOTIONALLY INVESTED
WHEN THEY SEE YOU GET
EMOTIONALLY INVESTED.
THEY APPRECIATE AND UNDERSTAND
YOUR EMOTION.
AND THEY KNOW THEY ARE AMONGST
FAMILY WHEN YOU INVEST YOUR OWN
EMOTION IN THAT.
SO IT'S IMPORTANT FOR YOUR OWN
FAMILIES, IMPORTANT FOR
INVESTIGATORS.
HE WAS AT THE FDA FOR A 3-HOUR
ROUNDTABLE DISCUSSION 10 DAYS
AGO.
ONE OF THE WORLD'S LEADING GENE
EDITORS CAME UP TO ME AFTERWARDS
AROUND REMINDED ME OF THE STORY
HOW HE GOT INVOLVED IN OUR
DISEASE BECAUSE HE SAW ME GET
EMOTIONALLY INVOLVED IN A
MEETING HE WAS IN AT THE NIH.
HE SAID I THINK I CAN PUT MY
SKILLS TO WORK IN FRIEDRICH'S
ATAXIA.
IT'S IMPORTANT AS AN INSPIRATION
FOR YOUR COMPANIES.
WE'VE HAD A NUMBER DO GRAND
ROUNDS.
THEY WILL STAND AT THE DOOR AS
YOU'RE LEAVING AND SAY NOW I
KNOW WHY I COME TO WORK EVERY
DAY, SO YOU DON'T GET THAT
INVESTMENT AND COMMITMENT
WITHOUT BEING EMOTIONALLY
AVAILABLE.
TO THE POINT HOW DO YOU KEEP UP
YOUR OWN ABILITY TO TELL YOUR
STORY, I WOULD SAY YOUR STORY IS
ALSO INSPIRING YOURSELF.
AND MY FINAL STORY, ONE OF THE
FINAL CONVERSATIONS I HAD WITH
OUR SON KEITH, HE KNOW HE HAD
ONLY DAYS TO LIVE.
HE KNEW HE HAD DAYS TO LIVE I
KNELT BESIDE HIM IN HIS ROOM, IN
HIS CHAIR.
I SAID I WANT TO THANK YOU AND
MOM FOR TRYING TO DO EVERYTHING
YOU COULD FOR ME.
I KNOW YOU THOUGHT YOU MIGHT BE
IN TIME FOR ME, IT DIDN'T QUITE
WORK OUT.
BUT I KNOW YOU'LL KEEP GOING
UNTIL YOU GET IT.
YOU'LL BE IN TIME FOR SOME OF MY
BUDDIES.
SO ABOUT A MONTH AFTER WE GOT
OUR FIRST APPROVED TREATMENT,
THIS WAS LIKE 15 YEARS AFTER WE
LOST OUR SON, WE WENT TO LUNCH
WITH TWO FAMILIES LOCALLY.
THEY WERE BOTH BUDDIES, PATIENTS
WERE BUDDIES, FRIENDS OF OUR SON
KEITH.
THEY HAD THEIR SKYCLARIS WITH
THEM AT THE MEAL.
YOU KNEW YOU WERE HONORING THAT
PLEDGE TO YOUR SON TO KEEP GOING
UNTIL YOU GET IT.
SO ...,
>> I'LL BE QUICK.
CHRISTINE CONDRA, THE B
FOUNDATION, WE LOST MY COUSIN
JENNY, A SISTER ON ERIN AND I,
ON CHRISTMAS DAY.
IT'S BEEN A JOURNEY WE NEVER
THOUGHT WE WOULD BE ON.
WE THOUGHT WE WOULD BE HAVING
KIDS TOGETHER.
WE THOUGHT WE WOULD BE HAVING
GLASSES OF WINE TOGETHER.
SHE WANTED TO MARRIED IN SONOMA.
WE LIVE IN THE PHILADELPHIA
AREA, OUR MOTHER WAS A
RESEARCHER AT NIH.
I WAS BORN IN D.C.
ONE THING THAT CONNECTED TO US
IS SOMETHING THAT'S REAL AND RAW
WE DON'T TELL OUR REAL STORY.
WE TELL THIS STORY, WE'VE BEEN
ON THE HILL FOR 11 YEARS
REPRESENTED PRO BONO, I'M HERE
ONCE A MONTH, MEETING WITH DR.
KOROSHETZ FOR 8 OR 9 YEARS.
I SPENT 10 YEARS IN BIOTECH AND
BIOPHARMA, ONCOLOGY AND
HEMATOLOGY, YOU GO IN, EVERYBODY
SAYS I'M SO SORRY, I'M SO SORRY.
YOU'RE LIKE, I'M GOOD, I'M GOOD,
I'M GOOD.
THAT ALSO TRANSLATES NOT IN
ALWAYS THE BEST WAY OF THERE'S
THIS BIT OF CALLOUSNESS TO US.
YOU'RE WALKING A FINE LINE
TELLING THE RIGHT STORY, OF
BEING EFFECTIVE BUT NOT SAYING,
WELL, YOU'RE THE WOUNDED BIRD IN
THE COCKTAIL PARTY.
EVERYONE IS LIKE, WAIT, WHAT DO
YOU DO?
AND KEEPING EVERYTHING POSITIVE.
ONE THING FOR US PEOPLE SAID
WHEN THEY GO TO OUR WEBSITE AND
WE HAVE AN ENTIRE "SHARE YOUR
STORY," CRAFTING THE RIGHT STORY
IS ALSO VERY IMPORTANT BUT WE
GIVE EVERYBODY HOPE.
STORYTELLING IS ONE OF THE
BIGGEST THINGS, THERE'S HOPE.
IT'S NOT JUST LOOKING AT
SOMETHING THAT'S TALKING ABOUT
THE MEDICAL SCIENCES, FOR US
EVERYTHING THAT COULD GO WRONG
WITH JENNY WENT WRONG, OUR
COUSIN THAT PASSED.
I'VE SAT IN CONGRESSMEN AND
WOMEN'S OFFICES, SENATORS, EVERY
WHERE, THEY ARE FLABBERGASTED
WHY WE'RE HERE IN 2025.
WE FOCUSED ON RESEARCH, OUR
PILLAR, HOW DO WE PREVENT THIS
TO THE POINT WHERE WE'VE LOOKED
AT SO MUCH OF THE ECONOMIC COST
ON THE HEALTH, PUBLIC HEALTH OF
ANEURYSMS, ASTRONOMICAL,
PRE-VERSUS POST-RUPTURE.
IN 2019 I WAS HERE, EVERY YEAR
I'M BLOWN AWAY, CONSTANTLY
WRITING, HAVE IDEAS FROM THIS, A
BIG CONVERSATION AT THE 4:00
SESSION WAS ABOUT HOW DO WE
ENGAGE RESEARCHERS.
AND HOW DO WE HAVE THESE YOUNG
RESEARCHERS FEEL A PART OF
EVERYTHING.
ONE OF THE BIGGEST THINGS WAS
THEY HAVE TO FEEL LIKE THEY ARE
PART OF A FAMILY, LIKE THEY ARE
PART OF -- I THINK IT WAS THE
MDS ORGANIZATION.
ONE THING WE HAD ALWAYS DONE AS
A RESEARCHER AT THE B FOUNDATION
THAT YOU THEN HAVE TO PRESENT --
YOUR AWARD IS PRESENTED BUT YOU
HAVE TO COME BACK THE FOLLOWING
YEAR AND GIVE ALL YOUR STUDIES.
THAT FAMILY COMES UP.
WE PRESENT NOW PICTURES OF THE
ENTIRE FAMILIES OF WHO THAT
GRANT IS NOW GOING TO, WHO IS
AFFECTED.
WE HAVE USUALLY ANYWHERE FROM
THREE TO FIVE GRANTS OF WHERE
THAT MONEY IS GOING TOWARDS.
AND THE FAMILIES ACTUALLY COME
UP WITH LOVED ONES, MOST
DECEASED, THIS IS WHO THIS WAS
FOR.
RESEARCHERS SAY I HAVE THIS ON
MY DESK, WHEN I COME BACK FROM
THE LAB I LOOK AT THE PERSON,
THAT'S WHAT I'M DOING.
SOMETHING WE'VE CREATED.
PEOPLE ARE PROUD TO BE IN THIS
THING, WE CALL IT THE HIVE.
WE HAVE OUR SAB, SOME TOP
VASCULAR NEUROSURGEONS IN THE
WORLD ON OUR SCIENTIFIC ADVISORY
BOARD, AND THE JUNIOR GUYS ARE
NOT JUNIOR AFTER 12 YEARS,
RISING UP AND TELLING THE
YOUNGER GENERATION, YOU HAVE TO
BE INVOLVED TO BE PART OF THE
FAMILY.
THAT'S A STORY IN AND OF ITSELF,
SOMETHING THAT WORKED REALLY
WELL FOR US TO CREATE THIS
INCLUSIVE FAMILY OF BEING A PART
OF SOMETHING AND MAKING A
DIFFERENCE SAVING LIVES FOR
PEOPLE.
THANK YOU. .
>> THESE STORIES HAVE BEEN
AMAZING.
IT'S HARD FOR ME TO SAY ANYTHING
IMPORTANT THAT HASN'T BEEN SAID,
EXCEPT TO REMIND EVERYONE THERE
ARE ALSO GOOD STORIES, STORIES
OF SUCCESS.
MY PERSONAL ONE I ALLUDED TO A
BIT THIS MORNING, HAVING A
GRANDSON WHO HAS BEEN
SUCCESSFULLY TREATED MUCH OF HIS
LIFE WITH A DRUG THAT I HELPED
DEVELOP.
WHEN I MEET WITH POLICYMAKERS,
WHEN I MEET WITH PEOPLE AT THE
STATE, FEDERAL LEVEL, THEY
UNDERSTAND THESE STORIES.
THEY DON'T UNDERSTAND BUDGETS.
THEY DON'T CARE TO HEAR ABOUT,
YOU KNOW, PEOPLE WHO DIE AND
THINGS, ALTHOUGH THEY REALLY
WANT TO HEAR THAT RESEARCH
ACTUALLY CAN PRODUCE SOMETHING.
WHEN I TELL THEM HOW THE GAUCHER
TREATMENT STARTED AT NIH,
DEVELOPED BY A COMPANY IN
BOSTON, AND HOW NIH-SUPPORTED
RESEARCH ON GENETIC
UNDERSTANDING OF GAUCHER ALLOWED
US TO DECIDE WHETHER OR NOT OUR
GRANDSON'S DISEASE WOULD AFFECT
THE CENTRAL NERVOUS SYSTEM, YOU
SEE, THEY UNDERSTAND THAT.
I THINK WHAT YOU SAID THIS
AFTERNOON IS VERY IMPORTANT.
AND I WISH MANY MORE OF MY
SCIENTIFIC COLLEAGUES COULD
LEARN TO TELL STORIES LIKE THIS
BECAUSE THAT'S WHAT WE NEED
RIGHT NOW WITH OUR LEGISLATURES
AND POLICYMAKERS TO MAKE THEM
REALLY UNDERSTAND, WHICH THEY
DON'T, HOW IMPORTANT THE
BIOMEDICAL RESEARCH IS THAT WE
DO.
AND IT'S THROUGH THESE STORIES.
>> I WOULD LOVE TO FOLLOW THAT
BECAUSE I THINK IT'S ABSOLUTELY
RIGHT THAT WE NEED TO COMBINE
OUR EMOTIONAL STORIES WITH THE
GOOD RESULTS THAT SOMETIMES
WE'RE ABLE TO ACCOMPLISH.
AND ANOTHER KEY AUDIENCE IN THAT
IS, AS THE LAST SPEAKER
INDICATED, CONGRESSIONAL STAFF
AND CONGRESSIONAL MEMBERS WHO
THERE'S NEVER BEEN A MORE
IMPORTANT TIME FOR US.
THERE'S A LIMIT TO WHAT WE CAN
SAY HERE TODAY GIVEN THE
AUDIENCE, OR OUR SPONSORS, BUT
THERE'S NEVER BEEN A MORE
IMPORTANT TIME FOR US TO
ADVOCATE.
PARTNERSHIP IS A TWO-WAY STREET.
LOOK AT WHAT NINDS AND NIH ARE
DOING FOR US.
WE NEED TO DO FOR THEM.
WE NEED ADVOCATE.
THEIR BUDGET IS UNDER SERIOUS
CONSTRAINT.
FRAUGHT WITH DANGERS OVER THE
NEXT SIX MONTHS.
SO, THERE'S NEVER BEEN A MORE
IMPORTANT TIME FOR US TO JOIN
OUR VOICES, JOIN OUR EMOTIONS,
TO ADVOCATE FOR THIS WONDERFUL
SCIENCE BEING FUNDED OUT OF THIS
INSTITUTE.
AND TO DO IT NOW.
OKAY.
LET'S GET TOGETHER, RAISE OUR
VOICES, TOGETHER WE'RE
UNCOMMONLY STRONG ALTHOUGH WE'RE
RARE.
>> LAUREN, ASSOCIATION FOR
FRONTOTEMPORAL ASSOCIATION.
I DIDN'T COME CLOSE TO THINKING
I WOULD SAY ANYTHING TODAY.
I FEEL STRESSED BY YOUR IDEA OF
STORY FATIGUE, I'VE BEEN THERE
AND DIDN'T KNOW I WAS THERE.
MY MOM PASSED OF FTD, AFFECTING
PEOPLE UNDER 60 LAST YEAR.
WHAT'S INTERESTING I WAS TELLING
HER STORY AND OUR STORY FOR SO
LONG BEFORE SHE PASSED BECAUSE
SHE COULDN'T SPEAK FOR HERSELF.
SHE LOOKED DIFFERENT, WOULD SHE
WANT HER PICTURE ONLINE, WOULD
SHE WANT REALITIES OF WHAT FTD
DID DISPLAYED?
IT'S MY EXPERIENCE BUT HERS TOO
AND WOULD AFFECT HER IMAGE.
WHAT DO I DO NOW THAT SHE'S
PASSED, FINDING A WAY TO
CONTINUE TO TELL HER STORY IN A
FIVE-MINUTE HILL MEETING IS
REALLY TOUGH BECAUSE IT'S THE
LITTLE THINGS THAT ARE MOST
IMPACTFUL.
THE 16th BIRTHDAYS, IT'S
TRYING TO GET MY DRIVER'S
LICENSE, I'M THE ONE THAT PUTS
EVERYBODY TO BED AT NIGHT IN THE
HOUSE BUT YOU CAN'T GET THERE.
I DON'T HAVE A SOLUTION PER SE
JUST TO VALIDATE THAT'S A
REALLY -- IT'S A REAL THING AND
DOES GIVE PEOPLE HOPE.
RIGHT NOW THERE ARE NO
INTERVENTIONS FOR FTD, NO CURES.
WHEN WE LOOK TO OUR COMMUNITY
AND WE'RE LIKE, OKAY, LET'S
GATHER COMPELLING STORIES, WHY
ARE WE GOING TO TAKE THE STORIES
FROM PEOPLE WHEN WE CAN'T GIVE
THEM ANYTHING IN RETURN?
WE CAN'T GIVE THEM HOPE YET.
RIGHT NOW WE HAVE A STRONG
COMMUNITY OF A FEW PEOPLE WITH
LIVED EXPERIENCE THAT ARE
TELLING THEIR STORIES AND
CAREGIVERS ARE TELLING THEIR
STORIES, PEOPLE LIKE ME, LIKE
YOU ALL, TELLING YOUR STORIES,
SUFFERING THROUGH THE STORY
FATIGUE BECAUSE PRETTY SOON
OTHER PEOPLE'S STORIES WILL
PROVIDE THAT MUCH MORE HOPE AND
REAL SOLUTIONS FOR PEOPLE.
>> VERY QUICKLY, FIRST OF ALL,
TOWARD YOUR POINT, AT THE
AMERICAN BRAIN -- SOCIETY FOR
NEUROSCIENCE LAST YEAR THE
AMERICAN BRAIN COALITION
ORGANIZED A SESSION ON
STORYTELLING, NOT FOR DISEASE
ADVOCATES BUT FOR
NEUROSCIENTISTS WHO SHOULD BE
ABLE TO TELL IN A SIMPLE WAY
WHAT THEY DO AND HOW THEY DO IT.
AND THEN THE SECOND POINT, I
MADE THIS POINT YESTERDAY AT THE
BRIEFING ON THE HILL, IT'S NOT
JUST POLICYMAKERS.
THEY HEAR US ALL THE TIME.
IT'S THE PERSON NEXT TO YOU IN
THE BAR, ON THE BUS, ON THE
PLANE.
WE ALL HAVE TO BE ADVOCATES FOR
THE IMPORTANCE OF SCIENCE AND
HOW IT AFFECTS HEALTH.
AND WE ALL HAVE TO TELL THOSE
STORIES NOT JUST TO MEMBERS OF
CONGRESS AND STAFF BUT TO
EVERYONE WE MEET.
LET THEM KNOW WHY THIS IS
IMPORTANT.
I WAS JUST SAYING TO KATIE AND
SHEILA WE HAVE AMERICAN BRAIN
COALITION PINS, WONDERFUL
BECAUSE SOMEONE WILL SAY WHAT
THE HELL IS THAT?
WE CAN SAY LET ME TELL YOU MY
STORY.
>> SUCH A POWERFUL SESSION.
I FEEL LIKE WE COULD HAVE HAD
EVERYONE IN THE ROOM UP HERE
WITH US.
THANK YOU FOR SHARING ALL OF
THAT.
ALL OF THE INPUT.
I TOOK A LOT AWAY FROM THAT.
IN CLOSING, I THINK REMINDER
THAT STORIES ARE NOT A SOFT
SKILL AS WE HEARD FROM EVERYONE
HERE TODAY.
THERE'S A STRATEGIC TOOL TO
DRIVE THE MISSION OF ALL OF OUR
WORK.
BUT MOST IMPORTANTLY THEY GIVE
US HOPE AND I THINK NOW IS NEVER
MORE IMPORTANT TIME TO SHARE
YOUR STORY, ENCOURAGE OTHERS AND
EMPOWER YOUR COMMUNITIES TO
SHARE STORIES.
THANK YOU SO MUCH.
>> THAT WAS REALLY FANTASTIC.
WE CAME FULL SWING, RIGHT?
STARTED WITH A POWERFUL STORY,
FROM THE CONGRESSWOMAN WEXTON
THIS MORNING, AND ENDED UP WITH
SOME GREAT STORIES HERE.
BUT EVERYBODY IN THE AUDIENCE
HAS THEIR OWN STORY.
AS A NEUROLOGIST, THERE'S A
NEUROLOGY BEHIND ALL THIS THAT
WE SHOULD THINK ABOUT.
A RECENT MEETING, WE ALL HAVE
THESE CIRCUITS IN OUR BRAIN
WHICH REALLY PRODUCE EMPATHY AND
COMPASSION.
BUT TO DEVELOP THAT YOU HAVE TO
BE EXPOSED TO SOMEONE WHO IS
SUFFERING.
SIMPLISTIC VIEW OF THINGS.
IF YOU DON'T PUT YOURSELF OUT
THERE, THE THINGS THAT DRIVE
MOTIVATION FOR PEOPLE TO HELP
DOESN'T HAPPEN.
SO, IT IS FATIGUING.
BUT WITHOUT IT, I THINK THINGS
DON'T GO ANYWHERE.
I THINK PHYSICIANS, AS A
PHYSICIAN WE SEE PEOPLE
SUFFERING ALL THE TIME, THAT
MOTIVATES THE HELL OUT OF US.
SCIENTISTS, IT'S IMPORTANT FOR
THEM TO HEAR THE STORIES SO THAT
THOSE EMPATHY AND COMPASSION
CIRCUITS KEEP THEM WORKING AT
NIGHT INSTEAD OF GOING HOME AND
HAVING A GLASS OF WINE.
SO ONE THING I WOULD SAY,
TECHNICALLY WE'VE BEEN TRYING TO
DO MORE OF THIS AT NINDS, SO
EVERY FRIDAY WE HAVE A TOWN
HALL, AND WE TRY TO GET SOMEONE
WHO HAS ONE OF THE DISORDERS TO
TELL THEIR STORY, SO ANYBODY
WANTS TO SIGN YOU WERE -- SIGN
UP WE'D BE HAPPY TO DO THAT.
IT'S USUALLY 10 MINUTES, A
VIDEO, IT'S A VIDEO LINK THING,
SO IT'S EASY TO DO.
YEAH, PEOPLE PUT VULNERABILITY
OUT THERE, A HUGE EFFECT ON OUR
STAFF.
AND THEN SPEAKING OF THE STAFF,
EVERYBODY TALKS ABOUT WALTER BUT
I DON'T REALLY DO ANYTHING.
I DO E-MAIL AND THAT'S IT.
BUT THERE ARE A LOT OF PEOPLE
HERE WHO WORK EVERY DAY, THE
STAFF OF NINDS, AND REALLY WANT
TO SAY I'M JUST THE FACE OF THE
ORGANIZATION BUT THEY DO THE
WORK.
COULD YOU ALL STAND UP FOR ONE
SECOND.
NINDS STAFF.
YOUR STORIES HAVE AN IMPACT ON
US FOR SURE.
THE ONLY THING THAT CAME TO MY
MIND AS YOU'RE TELLING THIS, IS
THAT THERE IS A COST TO
COMPASSION, THERE'S A COST TO
STORYTELLING.
YOU'RE PUTTING YOURSELVES OUT
THERE.
THE QUESTION IN MY MIND CAN YOU
WEAVE THE STORIES TOGETHER
SOMEHOW SO IT'S NOT ALL ON YOUR
SHOULDERS.
IT SEEMS LIKE THAT THERE'S SO
MUCH COMMONALITY HERE ONE COULD
THINK ABOUT DOING THAT SO THAT
IT'S NOT REALLY YOUR
RESPONSIBILITY DAY IN, DAY OUT,
BECAUSE THE STORIES, THEY HAVE
SPECIFIC THEMES TO YOUR
CONDITIONS, BUT TO THE BIG
AUDIENCE OUT THERE THEY HAVE SO
MANY MORE COMMONALITIES.
ONE THING TO THINK ABOUT.
I THINK THE MEDIA WHICH I DON'T
KNOW MUCH ABOUT, BUT THE SOCIAL
MEDIA, IT PROBABLY GIVES YOU
WAYS TO DO THAT, THAT WOULDN'T
HAVE BEEN POSSIBLE YEARS AGO.
SO, YEAH, WE WENT FULL SWING.
WE HEARD A LOT ABOUT THE HARD
CORE WAYS OF THERAPY
DEVELOPMENT, KNOWN PROFITS CAN
TAKE A ROLE, PERSONS WITH LIVED
EXPERIENCE IN THERAPY
DEVELOPMENT.
THE OTHER THING WE HEARD FROM A
GROUP OF I WOULD SAY FOLKS WHO
RUN NON-PROFITS, COLLABORATIVE
LIKE MILLIKEN, NATIONAL
ACADEMIES, AND THERE WE HAVE THE
OPPORTUNITY TO COME TOGETHER
AGAIN, WEAVE STORIES,
POTENTIALLY WORK MORE UNIFIED.
WHEN YOU GO TO CAPITOL HILL,
THEY ARE HEARING ONE PERSON
AFTER ANOTHER TELL A STORY, AND
IT'S NOT JUST THE NEURO PEOPLE
BUT ONE STORY AFTER ANOTHER, SOW
COMING TOGETHER WITH ONE VOICE,
THAT ABC TRIES TO PUSH, IS
REALLY IMPORTANT FOR ALL OF US.
THERE'S A LOT OF THE VALUE OF
WHAT YOU DO THAT CAN INCREASE IN
VALUE BY COMING TOGETHER THROUGH
THESE UMBRELLA ORGANIZATIONS
TOO.
SO, I THINK, RON, WE DID IT
AGAIN.
THIS YEAR WAS BETTER THAN LAST
YEAR.
WHAT CAN I SAY?
AND NEXT YEAR WILL BE BETTER
THAN THIS YEAR, SOMEHOW.
NOT SURE HOW.
BUT JUST FANTASTIC HOW PEOPLE
HAVE BEEN SO HONEST AND SO
GENEROUS WITH THEIR INTELLECT.
I THINK PEOPLE WALKING OUT OF
THE ROOM SHOULD HAVE A SENSE
THAT, YEAH, WE'RE ALL IN THE
SAME BOAT, ALL GOING IN THE SAME
DIRECTION.
AND, YOU KNOW, SOMETIMES YOU GET
SOME TOUGH SEAS BUT YOU'LL GET
THROUGH IT.
BOTH THE LINE OF PROGRESS WHICH
75 YEARS AGO WHEN NINDS WAS
STARTED WAS PRETTY DISMAL BUT
THE SUCCESS STORIES AS HARVEY
MENTIONED, RON MENTIONED, AND
OTHERS, THEY REALLY HAVE SHONE A
LIGHT AT THE END OF THE TUNNEL
FOR LOTS OF US.
SO WE'RE IN A GOOD POSITION AND
JUST GOT TO KEEP FIGHTING.
SO THANKS VERY MUCH TO THE
PANEL.
THANKS, EVERYBODY, FOR COMING
TOGETHER.
FEEL FREE TO CONTACT ME ANYTIME.
IF YOU HAVE FOLKS WHO WISH TO
TALK TO OUR STAFF, BE ON THEIR
FRIDAY MORNINGS, PLEASE LET US
KNOW.
ALL RIGHT.