WELCOME TO THE 16th NINDS
NON-PROFIT FORUM.
THIS IS ALWAYS ONE OF MY
FAVORITE DAYS OF THE YEAR WHERE
AT THE FORUM, WE PROVIDE AN
OPPORTUNITY FOR THE LEADERS OF
ALL THE DISEASE ORGANIZATIONS
AND OUR PORTFOLIO AND TO NETWORK
WITH THEMSELVES AND HAVE
DISCUSSIONS WITH NINDS STAFF AND
LEADERSHIP.
WE HAD HOPED TO RUN A HYBRID
EVENT BUT WE ARE IN MONTGOMERY
COUNTY SO THE DECISION WAS TO GO
VIRTUAL, HOPEFULLY NEXT YEAR WE
CAN COME BACK IN-PERSON.
WE HAVE OVER 240 REGISTRANTS FOR
THE EVENT AND WE WILL BE
RECORDING THE MAJORITY OF THE
SESSIONS THAT WILL BE AVAILABLE
AND TO LOOK WHAT THE WAS
DISCUSS.
NOW, EACH YEAR WE HAVE AN
EXECUTIVE COMMITTEE THAT WE WORK
WITH TO DETERMINE WHAT IS THE
THEME AND THE AGENDA FOR THE
MEETING AND THE AGENDA AND THEME
THIS YEAR IS HOW TO WORK WITH
QUOTE-UNQUOTE, ANYBODY FROM
INDUSTRY OR RESEARCH PARTNERS TO
NIH TO IMPROVE AND ADVANCE THE
RESEARCH TO GET BETTER
TREATMENTS FOR PATIENTS WITH
NEUROLOGICAL DISORDERS.
AND SO I WANT TO GO THROUGH A
COUPLE OF UPDATES FROM NINDS.
MISSION OF OUR INSTITUTE IS TO
SEEK FUNDAMENTAL KNOWLEDGE ABOUT
THE BRAIN AND THE NERVOUS SYSTEM
AND TO USE THAT KNOWLEDGE TO
REDUCE TH THE PEOPLE HERE KNOW
WE'RE UP AGAINST IN TERMS OF THE
CHALLENGES AND THERE'S A LOT OF
CHALLENGES AND MANY DIFFERENT
DISORDERS AND ALL OF WHICH CORE
A LOT OF SUFFERING AND TRAGEDY
FOR PEOPLE AND MOST OF THEM HAVE
LITTLE IN THE WAY OF REALLY
EFFECTIVE TREATMENTS.
WE HAVE SOME TREATMENTS AND SOME
CONDITIONS BUT IN GENERAL, WE'RE
STILL KIND OF SCRATCHING THE
SURFACE THERE SO RESEARCH IS
REALLY WHAT WILL LEAD TO BETTER
TREATMENTS ANDTREATMENT AND THAR
MISSION TO TRY AND GET US THERE.
AS I MENTIONED WHEN I STARTED,
THE FOREARM AND THE TOPIC AND
THE THEME OF PARTNERSHIP HOW TO
WORK WITH FOLKS FDA WAS SET BY
THIS COMMITTEE AND PEOPLE ARE
SEEING HERE ON THE LEFT AND FROM
THE DIFFERENT DISEASE
ORGANIZATIONS THAT REALLY SET
THE TONE AND THE PEOPLE ON THE
RIGHT AND THE NINDS PEOPLE WHO
WORK WITH THEM AND THEY TROY TO
PUT THIS SESSION, THESE SESSIONS
TOGETHER.
AND I DO HAVE ONE BITTERSWEET
ANNOUNCEMENT TO MAKE AND THAT IS
Dr. NINA SHORE, WHO HAS BEEN
THE DEPUTY DIRECTOR AT NINDS IS
GOING TO LEAVE NINDS AND INSTEAD
SHE'S GOING TO ANOTHER HIGHER
LEVEL MOVING TO LEAVE FOR ALL OF
NIH'S INTRAMURAL RESEARCH AND
SHE HAS BEEN THE ACTING SITE OF
THE DIRECTOR IN OUR INTRAMURAL
PROGRAM OVER THE LAST YEAR OR SO
AND WE'LL HAVE A GREAT PARTNER
AS WE MOVE INTO OUR NEXT PHASE
IN THE INTRAMURAL PROGRAM.
NOW, NINDS RECEIVES A BUDGET
FROM CONGRESS AND THAT BUDGET IS
THEN USED TO ADVANCE THE MISSION
AND I MENTIONED THIS BEFORE THAT
WE TRY TO HAVE MECHANISMS TO
FUND RESEARCH THAT GOES FROM THE
MOST BASIC RESEARCH AND ALL THE
WAY TO PATIENTS AND EVERYTHING
IN BETWEEN AND TRY VERY HARD NOT
TO LEAVE FUNDING GAPS WHERE
WHERE THERE'S NOT A MECHANISM TO
MOVE THINGS FORWARD TO GET TO
PATIENTS.
THAT BEING SAID, MOST OF OUR
FUNDING GOES INTO WHAT WE CALL
BASIC RESEARCH AND THAT IS 20%
TO 25% UNDER HOW THE BRAIN
WORKS, DISEASE AGNOSTIC AND
ABOUT 50% IS MECHANISMS OF
DISEASES SO, WHAT IS GOING ON IN
THE BRAIN AND PEOPLE WHO HAVE
EPILEPSIES OR PEOPLE THAT HAVE
DIFFERENT NEURODEGENERATIVE
CONDITIONS.
THAT'S BASIC RESEARCH AND THAT'S
WHERE MOST OF OUR MONEY GOES,
PROBABLY 75%.
THE REST IS SPLIT BETWEEN
TRANSLATIONAL RESEARCH WHICH IS
MOVING THINGS FROM THE BENCH TO
THE BEDSIDE THROUGH THE PATHWAY
THAT IS NECESSARY TO BRING A
THERAPY TO PATIENTS AND THAT IS
NOT TRIVIAL.
THERE ARE SAFETY CONCERNS, THERE
ARE LOTS OF STEPS THAT YOU HAVE
TO GO THROUGH JUST LIKE A
COMPANY WOULD TO MOVE THERAPY TO
PATIENTS THROUGH ALL THE STEPS,
GETTING IT AT THE END, APPROVAL
BY THE FDA TO TRY SOMETHING IN
PATIENTS AND SO-CALLED IND OR
IDE.
AND WE HAVE HERE AT NINDS ARE
LIKE A VIRTUAL PHARMACEUTICAL
COMPANY THAT CAN HELP PEOPLE
MOVE THINGS POINT TO THE IND FOR
THE FDA.
MANY OF OUR WORKS IN THE FARM
SIDE KALIN DUSTY AND I MUST SAY
MOST OF THE PEOPLE THAT WE WORK
IN THIS SPACE ARE GENERALLY TIED
TO SOME COMPANY, BIOTECH,
SOMETIMES A LARGER PHARMA
COMPANY AND OFTEN TIMES AN
ACADEMIC ASSOCIATED WITH A
COMPANY AND THIS IS WHERE WE DO
PARTNERSHIPS WITH OUR INGUS TREE
PARTNERS AND THE THEY'RE COMPARE
OR EFFECTIVENESS WHERE YOU TEST
TWO AGENTS THAT ARE AVAILABLE
AND BEEN APPROVED BY THE FDA AND
TO SEE WHICH ONE IS BETTER FOR A
PARTICULAR DISEASE AND WE ALSO
HAVE SO MOVING FROM THE IND
STAGE TO PEOPLE WOULD BE A
PHYSICIAN ONE STUDY TO SHOW IT'S
SAFE AND IN A PHASE 2 STUDY
LOOKING FOR SIGNS OF TARGETING
GAGEMENT OF PROOF OF PRINCIPLE
AND WE HAVE NETWORKS THAT WILL
DO THAT AND.
WE WOULD MUCH RATHER HAVE THE
INDUSTRY TAKE THAT OFF OUR
HANDS, BUT UNFORTUNATELY, FOR
MANY OF THE SORTERS IN OUR
PORTFOLIO, THE INDUSTRY ISN'T
READY AND THEY FEEL IT'S TOO
RISKY.
ALL ACROSS THE BOARD IN OUR
TRANSLATIONAL PROGRAMS AND OUR
EARLY TESTING, IN PATIENT
PROGRAMS, THE IDEA IS TO DERISK
THESE THERAPIES SO THAT INDUSTRY
WILL EVENTUALLY PICK THEM UP
BECAUSE WE DON'T MAKE DRUGS BUT
WE'RE TRYING TO DERISK THOSE
DRUGS FOR THE INDUSTRY.
IF YOU LOOK WHERE OUR MONEY
GOES, IT GOES TO INVESTIGATOR
INITIATE OR RESEARCH, THE DARK
BLUE PIECE.
THIS IS THE BEAUTY OF THE
AMERICAN SYSTEM, WHERE EVERYONE
CAN HAVE THEIR IDEA SENT INTO
NIH AND REVIEWED BY A STUDY
SECTION AND THE BEST IDEAS FLOAT
TO THE TOP AND IT GETS FUNDED
AND WE TEND TO BE VERY COGNIZANT
OF THE STUDY SECTIONS AND THE
CRITIQUES THEY PUT OUT, GUIDE
MUCH OF OUR FUNDING DECISIONS IN
THIS SPACE.
ANOTHER COLORS ARE PARTICULAR
AREAS THAT ARE FILLED IN SUCH AS
TRAINING OR SOME OF THE PROGRAMI
MENTIONED BUT THE MONEY GOES TO
INVESTIGATOR INITIATIVE
RESEARCH.
THERE ARE PRETTY COMPLICATED
MECHANISMS HERE TO KNOW WHAT IS
THE RIGHT MECHANISM FOR YOUR
TYPE OF RESEARCH?
WHETHER IT'S WITH AN INDUSTRY
PARTNER OR ACADEMIC PARTNER AND
WE'VE TRIED TO MAKE THAT
TRANSPARENT AS BEST WE CAN AND
THIS IS A PROGRAM WHERE WE HAVE
A PODCAST THAT GOES THROUGH ALL
THE DIFFERENT PROGRAMS WE HAVE
AND EXPLAINS HOW ONE MIGHT GET
INVOLVED IN ANY OF THESE
PROGRAMS.
THE NEXT SLIDE IS REALLY
COMPLICATED AND I MENTIONED
BEFORE THAT WE WANTED TO MAKE
SURE WE HAVE A FUNDING MECHANISM
FOR EVERY STEP THAT GOES FROM A
DISCOVERY STAGE TO AN ACTUAL
DRUG OR TREATMENT IN PATIENTS
AND SO WE HAVE PROGRAMS THAT ARE
WHAT WE CALL PRO CLINICAL
READINESS OR PROOF OF CONCEPT
PROGRAMS SO THESE ARE EARLY
STAGE PROGRAMS.
WE HAVE ACTUALLY CLINICAL TRIAL
READINESS GRANTS.
TO STUDY PATIENTS AND GETTING
UNDERSTANDING WHAT THE OUTCOME
MEASURE MIGHT BE FOR A COMING
CLINICAL TRIAL.
THEN WE HAVE THERAPEUTIC
DEVELOPMENT PROGRAMS AS THE
CLINICAL TRIAL READINESS AND WE
HAVE PHASE 1 IN HUMAN STUDIES
AND PHASE 2 FIRST AND TARGET
POPULATION AND PHASE 2 PROOF OF
CONCEPT, BIO MARKERRING AND
INFORM STUDIES AND THEN A PHASE
2 PRELIMINARY EFFICACY AND WE
RRARELY DOPHASE 3s.
WE DON'T DO ANY PHASE 4 POST
MARKETING BUT WE DO COMPARATIVE
STUDIES AND IMPLEMENTATION
STUDIES.
IT'S A LONG, COMPLICATED CHART
AND I'M FEELING IT IN HERE.
IT'S MUCH TOO COMPLICATED TO
REMEMBER.
THE KEY THING IS WE HAVE PROGRAM
DIRECTORS IN CHARGE OF THESE AND
THEY CAN GUIDE FOLKS TO KNOW AND
WHICH OF THESE PROGRAMS IS THE
BEST THING FOR WHAT YOU WANT TO
DO.
ALL THESE PROGRAMS IN THE
TRANSLATIONAL SPACE ARE WITH A
INDUSTRY PARTNER AND MUCH OF OUR
WORK IS FOCUSED ON SOME COMMON
DISORDERS SUCH AS EPILEPSIES OR
STROKE OR NEURO DEAGAIN NA TIVE
CONDITIONS AND WE HAVE THE
GREATEST NUMBER OF OUR DISEASE
ARE THE RARE DISEASES AND SO WE
HAVE A LARGE INVESTMENT IN RARE
DISEASE AND THE CONSORTIUM THAT
RUN OUT OF NCATS HAS MANY
NEUROLOGICAL CONSORTIUM WITHIN
IT AND WE FUND THESE
NEUROLOGICAL CON SORRY SUM HAS
PART OF THE RARE DISEASE
CLINICAL RESEARCH NETWORK AND
IT'S BEEN A VERY PRODUCTIVE
ENDEAVOR OVER THE YEARS.
THE TOPICS VARY AND THEY RUN A
PERIOD OF ABOUT FIVE YEARS AND
NEW GRANTS COME IN IN THIS RARE
DISEASE CONSORTIUM AND WE ARE
MOVING INTO THE SPACE OF
DEVELOPING GENE THERAPIES FOR
OUR RARE NEUROLOGICAL DISORDERS
AND MOST OF WHICH ARE RELATED TO
A GENETIC ABNORMALITY SO WE ARE
ONE OF THE PARTNERS IN THE
ACCELERATED MEDICINE PARTNERSHIP
WHICH IS RUN BY THE FOUNDATION
OF NIH AND WE'LL BE HEARING FROM
JULIE WHO IS THE DIRECTOR
SHORTLY AND SO THIS IS A
PARTNERSHIP WITH MULTIPLE
INDUSTRY AND MULTIPLE NON-PROFIT
ORGANIZATIONS AND THE IDEA IS TO
DEVELOP A PLATFORM THAT YOU CAN
PLUG IN DIFFERENT GENE THERAPIES
INTO A DELIVERY MECHANISM
USUALLY WHAT WE CALL A V VIRUS
THAT WOULD DELIVER THE GENE
THERAPY TO THE TISSUE AND
ORGANIZE OF INTEREST AND THIS
PROGRAM IS TO DEVELOP THAT KIND
OF PLATFORM AND STREAM LOIN THE
DREAM THEY WILL PEE DEVELOPMENT
PROCESS AND DO IT IN THE SAMEST
MASTER AS POSSIBLE.
A LOT OF IT HAS TO DO WITH
ADVANCING HOW THIS VIRUS
DELIVERS THE DIFFERENT JEANS AND
IN THE GENE THERAPY WORLD, THE
REAL CRUX IS IN THE DELIVERY
SIDE OF THINGS AND THE GOWNS
THEMSELVES ARE MORE EASILY
DEVELOPED AND UNDERSTOOD THAN
THE DELIVERY MECHANISMS AND JUST
ONE EXAMPLE IS A WORK THAT'S
BEEN COMING OUT OF THE BRAIN
INITIATIVE AND TO DEVELOP
VIRUSES THAT ARE TARGETING THE
BRAIN AND THE NERVOUS SYSTEM AND
THERE'S A BOONE BARRIER FROM
GETTING INTO THAT THE BRAIN AND
THAT IS AN ISSUE FOR THE VIRUSES
TOO WHICH LIKE TO GO TO THE LIGE
RIVER BUT THEY HAVE
SOPHISTICATED STREAMING THAT
WILL DELIVER THE GENES TO THE
BRAIN AND STAY AWAY FROM THE
LIVER WHERE YOU SEE THE TOXICITY
SO THIS IS REALLY EXCITING AND
FUTURISTIC BUT MAYBE NOT THAT
FAR AWAY.
IN NINDS WE DECIDED PARTICULARLY
UNDER THE LEADERSHIP OF Dr.
SHORE TO MAKE A BIG UNUSUAL THIS
AREA AND START AT THE ULTRARARE
GENE BASED THERAPY NETWORK
CALLED THE THIS IS TO DEVELOP
GENOMIC THERAPIES AND THEY CAN
BE NUCLEO TIDES AND THEY ARE
ANTI SENSE KNEW CLEO TIDES THAT
CAN BLOCK THE PRODUCTION OF A
HARMFUL GENE PRODUCT AND OR
VIRAL VECTOR BASED AND WE JUST
MENTIONED AND MAYBE EVEN GENOME
EDITING PROCESSES SO THESE NOW
THE ABILITY TO GO IN AND SNIP
OUT AWE MUTATION THAT IS CAUSING
TROUBLE AND REPLACE IT WITH THE
NORMAL THIS IS BEING DONE IN
SICKLE CELL DISEASE WHERE YOU
CAN TAKE OUT THE RED BLOOD
CELLS, EDIT THEM, WIPE OUT THE
GONE MARROW AND REPLACE IT WITH
THE EDITED PRO AGAIN TOE CELLS
AND CURE SICKLE CELL THAT WAY SO
THERE ARE A MATTER OF TIME
BEFORE WE WORKOUT HOW TO DO THIS
IN THE NERVOUS SYSTEM AND RIGHT
NOW, WE HAVE AND THESE ARE
USUALLY BIO AND THESE ARE
MANUFACTURE THE THE GREEN
PRODUCT AND TREAT PATIENTS AND
SO THAT IS OUR VERY AMBITIOUS
PLAN AND I THINK IT'S WE'RE JUST
BEGINNING SO YOU HAVE TO GO SLOW
AND CAREFULLY AS WE DO THIS AND
THIS IS WHERE I THINK THE FUTURE
LIES AND MANY OF OUR DISORDERS.
I DID MENTION GENE EDITING AND
JUST TALKING ABOUT THE URGENT
NETWORK AS SOMETHING THAT IS
POTENTIAL IN THE FUTURE AND I'M
ALSO CO DIRECTING A COMMON FUND
PROJECT DEVELOPING THESE TOOLS
THAT I MENTIONED WHICH CAN THEN
CUT OUT THE ABNORMAL GENE AND
THE NORMAL GENE AND USING THESE
REALLY ADVANCED GENOMIC
TECHNIQUES CALLED SAY CRISPR CAS
9 AND SO IN THIS COMMON FUND
PROJECT, THE FIRST FIVE YEARS,
WE FUNDED GROUPS TO DEVELOP
REALLY POWERFUL TOOLS AND THE
SECOND FIVE YEARS WE'RE HOPING
TO ACTUALLY TEST THOSE TOOLS AND
REFINE THEM BUT ALSO TO BEGIN,
IF SAVED, TO START TESTING THIS
THERAPY IN A COUPLE OF GENOMIC
DISORDERS BECAUSE THESE ARE
FIRST IN HUMAN TYPE OF WORK,
THESE WOULD BE DISORDERS THAT
ARE OTHERWISE FATAL IF NOT
TREATED AND WHERE THERE IS A
REAL ETHICAL FOUNDATION TO TAKE
A RISK WITH A TREATMENT SUCH AS
THIS.
AND THE HOPE IS WE LEARN HOW TO
DO THIS AND IT BECOMES SAFER
OVER TIME AND WE CAN REALLY
EXPAND, NOT JUST FOR CRISPR-CAS
9 BUT GONE THERAPY IN GENERAL TO
MANY OTHER DISORDERS AND SO IN
PHASE 2 WE'RE HOPING TO GET
THESE IN THE CLINICS.
IT'S A VERY AMBITIOUS PROGRAM.
WE TALKED A LITTLE BIT ABOUT OUR
THERAPY DEVELOPMENT GROUP, WHICH
I MENTIONED IS MADE UP OF VERY
SOPHISTICATED PROGRAM OFFICIALS,
MANY OF WHICH CAME IN FROM
INDUSTRY AND THEY RUN WHAT WE
CALL THE BLUE PRINT NEURO
THERAPEUTICS NETWORK AND THIS IS
ACTUALLY NOT JUST FOR NINDS BUT
OUR GROUP ENGAGES ACROSS ALL THE
NEUROSCIENCE INSTITUTES SO SOME
OF THE THERAPIES MIGHT BE FOR AN
EYE DISEASE OR DEMENTIA OR AGING
INSTITUTE OR A DISORDER IN THE
NATIONAL FOR CHILD HEALTH AND
DEVELOPMENT.
BUT THEY'RE ALL NEURO-BASED
THERAPIES AND THE IP BELONG TO
THE GRANTEE SO NIH IS NOT PART,
THERE'S NO RISK TO THE
INTELLECTUAL PROPERTY WE'RE JUST
FUNDING TO DERISK THE
THERAPEUTICS OF THE POINT OF THE
INDUSTRY INVESTMENTS AND
ADVANCES THE NEW DRUGS TOWARDS
PATIENTS EFFICIENTLY.
AND THERE'S VERY, AS I
MENTIONED, THERE'S PROGRAMS FROM
THE EXPLORATORY TO THE DRUG AND
AND I MENTIONED THESE ARE
GENERALLY THESE ARE LINED WITH
DISEASE ORGANIZATIONS AND MANY
OF THE INDUSTRY FOLKS ARE ALSO
ALIGNED WITH INDUSTRY PARTNERS
SO THESE ARE REAL EXAMPLES OF
THE PARTNERSHIP THAT WE'RE GOING
TO TALK ABOUT TODAY AND IN THE
BLUE PRINT THERAPEUTICS, THERE
ARE A SERIES OF CONTRACTS OUT TO
COMPANIES MOST BIO TECHS DO HAVE
THAT AND MOST OF THE AK DEEMIA
DON'T HAVE THE CHEM ASSIST AND
PEOPLE WHO DO TOXICOLOGY AND
MANUFACTURING.
SO, WE HAVE CONTRACTS OF THE
PEOPLE WHO WIN THE AWARDS HAVE
ACCESS THOUGH THESE CONTRACTS TO
DO THIS WORK AS THEY MOVE
TOWARDS THE IND.
AND I'M GOING TO END NOW JUST
WITH A MENTION ABOUT OUR
TRAINING PROGRAMS AND BECAUSE
FOR MY VIEW, NINDS, NIH HAS
RESOURCES.
THERE ARE A HOST OF REALLY GREAT
IDEAS AND OPPORTUNITIES.
SO, WHAT IS IT THAT REALLY SETS
THE FUTURE AND THE PACE OF THE
DISCOVERY AND TRAPS LAYING AND
THAT IS ACTUALLY PEOPLE.
I THINK THAT THE BOTTLENECK IS
REALLY THE PEOPLE IN THE PROCESS
OF TRANSLATION.
THERE ARE SOME GOOD ONES, BUT
IT'S NOT THE MOST COMMON CAREER
PATH FOR MANY PEOPLE.
MANY PEOPLE WHY GREAT SCIENTISTS
WHO LATE IN THEIR CAREER DECIDED
THEY WANTED TO DEVELOP THEIR
IDEAS INTO A THERAPY.
THEY'RE STILL NAIVE AT THAT
POINT SO WE REALLY NEED TO DO IS
TO WORK ON TRAINING PEOPLE IN
THIS SPACE AND WE HAVE A
TRAINING PROGRAM NOW FOR
TRANSLATIONAL RESEARCH AND I
THINK IT'S THE DISEASE
ORGANIZATIONS THAT CAN REALLY
HOOK SOMEONE ON THIS IDEA OF
WORKING TOGETHER WITH YOU TO
DEVELOP THERAPIES FOR YOUR
PATIENTS AND FOR THE PEOPLE THAT
YOU REALLY CARE ABOUT AND I
THINK YOU ARE MUCH MORE
CONVINCING THAN THE GOVERNMENT
TO SAY I'M GOING TO WORK ON THIS
RARE DISEASE FOR THE REST OF MY
CAREER.
IT'S SOMETHING THAT NIH WILL
MAKE HAPPEN BUT DISEASE
ORGANIZATION, YOU CAN MAKE IT
HAPPEN.
THE NUMBER OF APPLICATIONS HAS
BEEN FLAT GOING BACK 22 YEARS
AND IF YOU AT THE STAGE OF
TRANSLATION AND SO AGAIN, I
THINK IT'S A DISEASE
ORGANIZATIONS THAT CAN REALLY
LIGHT THE FIRE AND FOLKS TO WHO
ARE TRAINED TO TAKE CARE OF
PATIENTS AND SPEND THEIR CAREER
WORKING WITH PATIENTS TO DEVELOP
THERAPIES BUT IT'S ONE OF THE
BIGGEST BOTTLENECKS WE HAVE.
IN TERMS OF HOW THE NINDS
PARTNERS, WE ARE ALWAYS LOOKING
TO PARTNER.
WITH INDUSTRY THAT CAN ADVANCE
OUR MISSION WITH DISEASE
ORGANIZATIONS AND WE HAVE OUR
COUNCIL THAT MAKES ALL THE
DECISIONS ON WHAT WE FUND.
WE DON'T FUND ANYTHING UNLESS
THE COUNCIL GROWS AND THIS HAS
PUBLIC MEMBERS AS WELL AS
SCIENTISTS AND PHYSICIANS.
WE DO STRATEGIC PLANNING WHICH
Dr. SHORE LED FOR US BUT RIGHT
NOW WE'RE DOING STRATEGIC
PLANNING AND ALL THESE DIFFERENT
EVENTS COME UP, WE'RE ALWAYS
PARTNERING WITH THE DISEASE
ORGANIZATIONS AND WE'RE ALSO
LOOKING FOR INDUSTRY INPUT
BECAUSE WHEN YOU GET TO THE
TREATMENTS BECAUSE THAT'S WHAT
IS REALLY GOING TO MAKE A
DIFFERENCE FOR PATIENTS AND WE
HAVE CONFERENCES WHERE THESE ARE
ALWAYS INCLUSIVE OF PUBLIC
MEMBERS AND THOSE WHO REPRESENT
THE DISORDERS THAT WE'RE TRYING
TO ATTACK.
IT'S A LITTLE BIT ABOUT HOW WE
PARTNER AND THE MECHANISMS FOR
PARTNERSHIP AND I'M LOOKING
FORWARD TO THE REST OF THE
DISCUSSIONS WITH REGARDS TO THIS
TOPIC AND HOW WE WORK WITH
INDUSTRIES AND NIH AND ACADEMIC
TO ADVANCE OUR INDIVIDUAL
MISSIONS.
I WANT TO THANK YOU FOR YOUR
ATTENTION AND NOW I'D LIKE TO
INTRODUCE OUR KEYNOTE SPEAKER,
WHO IS Dr. -- I HAVE MY NOTES
HERE.
Dr. JULIE GERBERDING THE NEW
CHIEF OFFICER FOR THE FOUNDATION
OF THE NATIONAL INSTITUTE OF
HEALTH SO OTHERWISE KNOWN AS
FNIH AND THE FOUNDATION WAS SET
UP BY CONGRESS TO MANAGE
RESEARCH ALLIANCES WITH PUBLIC
AND PRIVATE INSTITUTIONS AND
SUPPORT OF THE MISSIONS OF THE
NIH AND SO IT IS REALLY A
PARTNERSHIP ORGANIZATION AND
JULIE COMES WITH EXPERIENCE AND
CREDENTIALS AND PASSION FOR THIS
TYPE OF PARTNERSHIP.
SHE WAS PREVIOUSLY THE EXECUTIVE
VOICE PRESIDENT OF THE CHIEF
PATIENT OFFICER AT MERK AND SHE
JOINED MERK IN 2010 AS A
PRESIDENT OF THE THEIR VACCINE
PROGRAM AND DIRECTOR OF THE CDC
AND SHE LED THE AGENCY BETWEEN
2002 AND 2009 AND SHE'S TRAINED
PHYSICIAN.
SHE DID HER UNDERGRADUATE MD
DEGREES AT CASE WESTERN AND
TRAINED IN INTERNAL MEDICINE AND
INFECTIOUS DISEASE AT UNIVERSITY
OF CALIFORNIA SAN FRANCISCO AND
SHE WAS ON THE FACULTY THERE FOR
MANY YEARS AND AND ALSO AND
UNIVERSITY OF CALIFORNIA
BERKELEY AND SHE'S REALLY VERY
WELL RESPECTED AND MULTIPLE
HONORS AND AWARDS AND WE ARE SO
LUCKY TO HAVE JULIE RUNNING THE
FOUNDATION FOR NIH AND NINDS HAS
HAD SO MANY GREAT RELATIONSHIPS.
I'M LOOKING FORWARD TO YOUR
COMMENTS.
>> THANK YOU, WALTER.
I'M REALLY HONORED TO BE THE
KEYNOTE OF THIS PARTICULAR
PROGRAM AND IT'S REALLY PROBABLY
MY FIRST PUBLIC PRESENTATION ON
BEHALF OF FNIH AND CONJUNCTION
OF THE NIH AND I COULDN'T THINK
OF A BETTER AUDIENCE FOR IT SO
IT'S TEAR TO MY HEART TO REALLY
BE ENGAGING WITH PATIENTS
ORGANIZATIONS, CARETAKER
ORGANIZATIONS, ET CETERA, BUT
PARTICULARLY WITH A PHOTOGRAPHIC
HE IS I HAVE TO ADMIT TO YOU I
WASN'T REALLY FAMILIAR WITH THE
FNIH EVEN WHEN I WAS IN
GOVERNMENT AND NOW THAT AND THE
ROLE THIS ORGANIZATION PLAYS AND
TRYING TO BE A INTERFACE NOT JUT
BETWEEN THE NIH AND INDUSTRY
THAT YOU'VE BEEN HEARING ABOUT,
BUT WITH OTHER ORGANIZATIONS
INCLUDING A NUMBER OF PATIENT
AND NON PROFITS AND THE GATES
FOUNDATION, OTHER FOUNDATIONS,
ET CETERA, AND AS WELL AS JUST
INDIVIDUAL STAKEHOLDERS AND
SOMETIMES PHILANTHROPISTS WHO
ARE INTERESTED IN OUR WORK OUR
MISSION IS VERY BROAD.
IT'S REALLY TO SUPPORT THE
MISSION OF THE NIH SO THAT IS A
VERY BROAD INTERPRETATION OF OUR
CONGRESSIONAL AUTHORITY BUT WE
DO TEND TO FOCUS ON THREE MAIN
CHANNELS OF ENGAGEMENT.
ONE IS SUPPORT FOR SCIENCE AND
WE'LL SAY A BIT MORE ABOUT THAT
AND THE SECOND FOR SUPPORT FOR
SCIENTISTS AND WE DO THAT BY
SUPPORTING LABS AT THE NIH,
SCIENTIFIC MEETINGS, ET CETERA,
ET CETERA, TO TRY TO NOT ONLY BE
A FORCE OF THE ACTUAL RESEARCH,
BUT SUPPORTING THE PEOPLE AND
THE PIPELINE OF PEOPLE NECESSARY
TO CONDUCT AND PARTICIPATE IN
THAT RESEARCH IN AND OUT OF THE
NIH.
IN OUR THIRD DOMAIN, WE HAVE A
ROLE IN THE THOUGHT LEADERSHIP.
FOR ME COMING INTO THIS ROLE,
WITH MY BACKGROUND AS THE CHIEF
PATIENT OFFICER AT MERK, I
REALLY WANT TO BRING IT'S
EXCITING FOR ME TO REALLY
INITIATE THIS CONVERSATION AND
I'M SHARING MY E-MAIL AT THE END
OF IT BUT I DO HOPE TO BE ABLE
TO MEET WOMAN OF YOU FOR SEVERAL
REASONS OF HOW WE MIGHT BE
HELPFUL IN THE FUTURE AND I'M
INTERESTED UNTIL THE PATIENT
ENGAGEMENT AND THE END-TO-END
CLINICAL RESEARCH PROCESS.
SOMETHING I'VE LEARNED ABOUT
FROM THE INDUSTRY SIDE BUT I
WOULD LIKE TO LEARN MORE ABOUT
IT FROM THE EXTERNAL
PERSPECTIVE.
I'VE MEET YOU AT THE BIO ANNUAL
PATIENT ENGAGEMENT SUMMIT, AND I
HOPE I WILL BE INTERACTING WITH
SOME OF YOU IN MY ROLE AS A
MEMBER OF THE NATIONAL HEALTH
COUNCIL BOARD SO I LOCK FORWARD
TO LINING, LEARNING AND SEE WHAT
FNIH CAN DO TO BE INCLUSIVE OF
THE POPULATIONS WE'RE ALL TRYING
TO SERVE.
I DON'T KNOW WHAT WAS IN THE
MINDS OF CONGRESS WHEN THEY
CREATED THE FNIH BUT PART OF
THAT EFFORT WAS AN ATTEMPT TO
BRING AN INTERFACE IN THE SENSE
MIDDLE WEAR SO THAT THE
GOVERNMENT AND INDUCE TREMENDOUS
INDUSTRY CANCOLLABORATION.
IT'S NOT POSSIBLE FOR INDUSTRY
TO GIVE MONEY TO THE GOVERNMENT
OR KIND OF OBVIOUS CONFLICT OF
INTEREST REASONS.
SO IT'S IMPORTANT TO HAVE A
ADJUDICATED, ETHIC-BOUND,
MECHANISM SO THAT APPROPRIATE
COLLABORATIONS CAN BE CRAFTED
AND ALSO THEY CAN BE CONDUCTED
IN THE RIGHT WAY.
AND I WON'T GO INTO SOME OF THE
COMPETENCIES OF THE FNIH BUT
I'LL HIGHLIGHT A COUPLE THAT
STAND OUT TO ME.
ONE IS PROJECT MANAGEMENT AND
I'LL USE ACTIVE AS AN EXAMPLE OF
THAT.
THE MECHANISMS THAT ARE BROUGHT
ALL KINDS OF PARTNERS TOGETHER
TO ACCELERATE THE PROGRESS OF
ANTI VIRALS AND VACCINES AND
DIAGNOSTICS DURING COVID.
THESE PROJECT MANAGEMENT, KIND
OF IN THE BACKGROUND BUT THE
BEST PROJECT MANAGEMENT I'VE
SEEN IN ANY SETTING, INCLUDING
MY ACADEMIC BACKGROUND AND
GOVERNMENT BACKGROUND AND INDUCE
TREMENDOUS BACKGROUND AND THIS
IS JUST A BRILLIANT SET OF
SCIENTISTS AND THEIR AND CREATE
A WHOLE GREATER THAN THE SOME OF
THE PARTS.
ONE OF THE STRENGTHS REALLY THE
INTERFACE WITH THE FDA AND THE
ABILITY TO WHICH IS REALLY
IMPORTANT TO INDUSTRY WHEN
THEY'RE STRIKING OUT TO HAVE
CLARITY ON WHAT THE FDA WILL
CONSIDER AS THE KIND OF END
POINT OR THE DAY THAT THAT WOULD
HELP FACILITATE DECISIONS AROUND
DRUG APPROVAL AND LABELING.
SO ON THE NEXT SLIDE, JUST
TALKING A LITTLE BIT ABOUT THE
BIG NUMBERS, TODAY, THE
FOUNDATIONS JUST OVER
25-YEARS-OLD, AND IT HAS RAISED
ABOUT 1.2 BILLION IT'S MORE IF
WE COUNT SOME OF THE ACTIVE
RESOURCES AND WE'VE RAISED A
SUBSTANTIAL INVESTMENTS AND
WE'RE PROUD OF THE FACT THAT THE
VAST MAJORITY OF THOSE DOLLAR
REALLY DO GO DIRECTLY TO SUPPORT
PROGRAMS AND ABOUT 600 OF THEM
TO BE EXACT.
AND THE ACTIVE RESEARCH PROJECTS
WE HAVE GOING ON RIGHT NOW ARE
117 OF THEM, WHICH I THINK IS A
TRIBUTE TO THE SMALL FOUNDATION
AND WE HAVE PEOPLE WHO ARE VERY
ASSERTIVELY INVOLVED IN
SUPPORTING AND I'VE DEFINED HOW
WE DECIDE WHAT PRIVATE-PUBLIC
PARTNERSHIP MAKES SENSE TO US
AND WE HAVE A GOVERNANCE
STRUCTURE THAT HELPS ADJUDICATE
THESE DECISIONS AND WE PUT A
GREAT DEAL OF EFFORTS ON POLICY
AND COMPLIANCE SO WE ARE NOT INA
SITUATION WHERE WE VIOLATE THE
PRINCIPLES OF ETHICAL CONDUCT OR
CONFLICT OF INTEREST AND WE
REALLY OF COURSE ENDORSE AND
SORT POLICIES OF THE NIH AS WELL
IN THAT REGARD.
OUR FUNDRAISING AND RELATIONSHIP
MANAGEMENT IS A VERY IMPORTANT
PART OF OUR ORGANIZATIONS AND WE
ARE PERMIT TODAY SOLICIT FROM
FOUNDATION AND FROM
TRANSFORMATIONAL DONORS AND FROM
GRATEFUL PATIENTS AND TIMES
WE'RE NOT ALLOWED TO SOLICIT
FROM THEM DIRECTLY OF COURSE BUT
WE DO HAVE OPPORTUNITIES TO
RESPOND TO THEIR INTERESTS AND
SOME OF THAT INTEREST HAS LED TO
FUNDING OF CERTAIN HAB TORIES OR
CERTAIN INVESTIGATORS OR
FELLOWSHIPS AT THE NIH SO THAT'S
PART OF OUR SUPPORTING
SCIENTISTS COMPONENT OF WHAT WE
DO AND I MOVE TO -- I WANT TO
MAKE A POINT HERE ABOUT THE LAST
ITEM ON THE SLIDE.
OUR MECHANISMS OF ENGAGEMENT IS
IN THE PRE COMPETITIVE SPACE.
SO WE DON'T BRING TWO PHARMA
COMPANIES TOGETHER AND SHARE
STRAYED SECRETS OR CREATE A
SITUATION THAT WOULD BE
PERCEIVED AS ANTI COMPETITIVE.
WE DO OUR WRACK IN THE MORE
SCIENTIFIC PRE COMPETITIVE
ENVIRONMENT AND IT ALLOWS PEOPLE
TO BE OPEN WITH THEIR
INFORMATION AND THEIR DATA AND
THE MECHANISMS FOR HANDLING
INTELLECTUAL PROPERTY ARE WORKED
OUT BEFORE ANY PROJECTS BEGIN
AND I THINK THIS THIS IS PLACE.
WE HAVE A GOOD TRACK RECORD MAN
NOT AGING WITH THE ETHICAL
FRAMEWORK AND ON THE NEXT SLIDE,
I HAVE HOW THIS PLACE OUT IN
REAL-TIME AND ANYONE CAN COME UP
WITH A REASON TO HAVE A
COLLABORATIVE RESEARCH PROGRAM
INCLUDING GOVERNMENT INDUSTRY
AND AK A DEM I GUESS AND
ESPECIALLY HIGHLIGHT PATIENTS
GROUPS AND YES SOME OF THE
PATIENT ORGANIZATIONS THAT ARE
PARTICULARLY INTERESTED IN
CLINICAL RESEARCH ARE A GREAT
SOURCE OF IDEAS AND CONNECTIONS
THAT HELP BUILD THE CRITICAL
MASS OF PEOPLE COMING TOGETHER
TO FORMULATE A SPECIFIC IDEA AND
THE NIH PROCESS WHERE INSTITUTES
ARE THE NIH DIRECTOR SEES THE
OPPORTUNITY FOR CROSS CUTTING
PROGRAMS OR THE AT ANY TIME A
COLLABORATION CAN CREATE AND
THERE'S AN INTERNAL PROCESS AT
NIH TO MAKE SURE THAT THE
PROPOSAL MEETS NIH CRITERIA AND
ULTIMATELY THEY END UP AT THE
FNIH AND WE HAVE A ROBUST PANEL
OF SCIENTIST AND OUR BOARD OF
DIRECTORS WHO INCLUDE
WORLD-CLASS SCIENTISTS TO REVIEW
THE PROPOSALS AND MAKE DECISIONS
ABOUT WHETHER IT'S A GO OR A
NO-GO PROGRAM.
IF IT'S A GO PROGRAM, WE CREATE
THE GOVERNANCE, RESTORE THE
PARTNERSHIP AS QUICKLY AS WE
CAN, WHICH SOMETIMES ISN'T
QUICK.
WE RAISE THE MONEY IN ORDER TO
INITIATE.
ONE OF OUR PRINCIPLES BECAUSE WE
ARE USING OTHER PEOPLE'S MONEY
TO FUND THIS RESEARCH WE GET US
TO THE FIRST MILESTONE SO WE
HAVE BUT ULTIMATELY THIS PROCESS
RESULTS IN A PROGRAM THAT'S BEEN
VETTED AND TO HIT THE GROUND
RUNNING AND TO MOVE THINGS
FORWARD NOT ALL PARTNERSHIPS
MAKE IT.
FIRST AND FOREMOST WE MAKE SURE
WITH WHAT WE'RE DEALING WITH IS
SOMETHING THIS ADDRESSES
IMPORTANT UNMET NEEDS AND
SOMETHING THAT HAS A HYPO TEN
SHALL FOR TRULY IMPACTING
PATIENTS AND MAKING A DIFFERENCE
FOR THEM AND THAT IS A REALLY
IMPORTANT AREA WHERE THE CONCEPT
OF EQUITY COMES IN AND OUR
WEIGHTED PROBLEMS AND NOT
NECESSARILY PROBLEMS THAT
ADDRESS ALL COMMUNITIES AND WE
ARE GOING TO INCREASE OUR
EMPHASIS ON PROGRAMS THAT
ADDRESS HEALTH EQUITIES AND MAKE
SURE IT'S ONE OF THE SPECIFIC
CRITERIA WHAT WE CONSIDER AS AN
OPPORTUNITY TO AND THE OTHER
CRITERIA ARE A PRETTY OBVIOUS
HERE AND LOCK AT NUMBER FOUR
BECAUSE SOMETIMES WE HAVE A
FANTASTIC IDEA AND WE JUST CAN'T
GET IT FUNDED AND THEY'RE JUST
ISN'T ENOUGH INCENTIVE FROM THE
INDUSTRY PARTNERS AND NIH HAS
COMPETING PRIORITIES AND THIS IS
WHERE WE'RE GOING TO GO FORWARD
AND HOW WE CAN RAISE MONEY IN
OTHER DOMAINS THAT WOULD HELP
GIVE US AT LEAST THE START UP
RESOURCES TO TEST IDEAS OR PILOT
IDEAS AND TO MAKE SURE THAT WE
HAVE PROOF OF CONCEPT BEFORE WE
LULAUNCH INTO A MORE AGGRESSIVE
FUNDRAISING OPPORTUNITY.
THIS SHOWS YOU THAT, AS
RESOURCES BECOME AVAILABLE, SOME
OF THAT MONEY GOES TO THE NIH
INSTITUTES WHO ADDED TO THEIR
OTHER PROGRAMMATIC MONEY AND
MOVE IT OUT TO THE PEOPLE THAT
ARE GETTING FUNDING THROUGH THE
MANY MECHANISMS THAT WALTER JUST
SHOWED US AND THAT HAPPENS
THROUGH THE USUALLY CUSTOMARY
NIH MECHANISMS BUT FNIH ALSO
PROVIDES GRANTS TO SCIENTISTS
AND WE HAVE CONTRACTS WITH
VARIOUS RESEARCH ORGANIZATIONS
AND WE SUPPORT DATA COORDINATING
CENTERS AND OTHER KINDS OF
INVESTIGATORS TO PARTICIPATE IN
THE COLLABORATION.
ALL OF OUR WORK IS DONE IN AN
OPEN PLATFORM MEANING THE DATA
ARE SHARED AS PART OF THE
ADVANTAGE TO THIS BEING PRE
COMPETITIVE THAT WE HAVE THE
OPPORTUNITY TO MAKE WHAT WE'RE
DOING VISIBLE SO OTHERS CAN
LEARN FROM IT AND AND I HAVE A
WONDERFUL EXPERIENCE AND RELATED
TO THE CONSEQUENCES OF REPEATED
EXPOSURES TO CORONAVIRUS
VACCINES AND TO TALK AND SHARE
THIS ELEGANT WORK.
IT WAS JUST REFRESHING TO ME TO
PARTICIPATE OPEN EXCHANGE AND
SCIENTIFIC COMMUNICATION WASSEN
LIGHT EPING AND I THINK THAT
VERY INDICATIVE OF THE KIND
ATMOSPHERE AND WE TRY TO AND THE
NEXT SLIDE, JUST TO GIVE YOU A
LITTLE BIT.
RIGHT NOW WE HAVE $445 MILLION
IN PROGRAMS IN PROGRESS.
SO THAT IS A LOT OF PROJECTS
THAT ARE GOING ON AND THESE ARE
BIG PROJECTS.
YOU'VE HEARD WALTER MENTION A
COUPLE OF THEM.
THE FOUR BIG CATEGORIES FOR US
RIGHT NOW ARE THE SO-CALLED AMP
PROGRAMS.
AND KIND OF AN ACCELERATED
OPPORTUNITY TO DEFINE NEW
TARGETS OR NEW APPROACHES TAG
EIGHT INDICATION AND MEDICATIONS
THAT WOULD SELECT THAT TARGET
AND THOSE PROGRAMS, THERE ARE
EIGHT OF THEM RIGHT NOW THEY ARE
LONG STANDING AND THEY HAVE A
VERY WONDERFUL TRACK RECORD OF
CONTRIBUTION AND THE OTHER
LONGSTANDING PROGRAM IS THE
BIOMARKERS PROGRAM AND THIS IS A
PROGRAM THAT LOOKS FOR THE
PROGNOSTIC BIOMARKERS THAT HELP
DETERMINE WHO IS AT RISK FOR
DISEASE PROGRESSION, FOR
EXAMPLE, SOMETHING LIKE NON
ALCOHOLIC LIVER DISEASE WHERE
IT'S IMPORTANT TO KNOW WHO IS
MOST LIKELY TO PROGRESS TO
CIRRHOSIS SO THAT THOSE ARE THE
PEOPLE THAT YOU WOULD WANT TO
FOCUS INTERVENTION ON AND NOT
FORCE OTHER PEOPLE TO HAVE A BUY
ON SEE OR UNNECESSARY INVASIVE
PROCEDURES BECAUSE THEY'RE NOT
GOING TO BE AT RISK FOR
PROGRESSION.
IF YOU WERE STUDYING A PROBLEM
WITH A LONG LIFELINE, THE
STUDIES WOULD HAVE TO GO ON
FOREVER TO SHOW HARD
DEMONSTRATIONS OF DECREASED
MORTALITY OR DECREASED LONG-TERM
MORBIDITY AND IF YOU HAVE A
SURROGATE BIOMARKER THAT IS
HIGHLY CORRELATED WITH THOSE END
POINTS AND YOU HAVE THE RIGHT
DATA ON THE RIGHT
STANDARDIZATION TO USING THAT
END POINT YOU CAN CONDUCT
CLINICAL TRIALS AND GET DRUG
APPROVAL BASED ON THAT SURROGATE
MARKER OF SUCCESS AND THAT'S
SOMETHING THAT IS SCIENTIFICALLY
SIMPLE MINDED BUT REALLY HARD TO
DO AND -- WHICH ARE FOUR
CATEGORIES OF PROGRAMS THAT ARE
PRIMARILY FUNDED THROUGH THE
GATES FOUNDATION AND PARTNERS
THAT ARE GLOBAL PORTFOLIO AND I
JUST WANT TO MAKE THE POINT THAT
WE'RE NOT TOTALLY FOCUSED ON THE
DEVELOPED WORLD AND WE'RE
THINKING ABOUT HOW WE CAN BRING
THE PROCESS NO A EQUITABLE
MANNER ACROSS ALL OF OUR DISEASE
CATEGORIES AS WE EVOLVED THE
FNIH.
NEXT SLIDE, PLEASE.
AND I THINK WE CAN JUST SKIP
THOSE AND I'VE MENTIONED THESE
BEFORE WELL MAYBE JUST QUICKLY
TO SHOW YOU THE SCALE OF
INVESTMENTS IN THESE VARIOUS
PROJECTS SO FOR EXAMPLE, I
MENTIONED THE BIOMARKERS
CONSORTIUM AND WE HAVE THE
INVESTED IN THAT AND IT PAYS FOR
IMAGING STUDIES THAT MIGHT NOT
BE DONE IN THE COURSE OF A
CLINICAL TRIAL OR A CLINICAL
INVESTIGATION TO DETERMINE IF
SOMETHING IS AN EFFECTIVE
BIOMARKER OR NOT SO, THREE
MOVEMENTS ABOUT WHAT IS THE
STANDARD DATA SET AND HOW WE CAN
POOL THIS TOGETHER TO ACHIEVE
THE KIND OF STATISTICAL POWER
THAT WOULD HE CAN SELL RATE OUR
DECISION-MAKING.
NEXT SLIDE, PLEASE.
I'M GOING TO END WITH A LITTLE
BIT OF IN-HOUSE PERSPECTIVE
BECAUSE I AM DEFINITELY STILL IN
THE LEARNING CURVE AND I'M NEW.
WE WE HAVE THE SAME CHALLENGES
THAT BIG ORGANIZATIONS HAVE AND
AND I'M NOT SURE MANY PATIENTS
INCLUSION AND NOT JUST IN TERMS
OF WHO WE ARE AND IN THE WORK WE
DO SO I'M PLEASED SO SAY THE
BIOMARKERS CONSORTIUM SPENT A
EXECUTIVE COUNTY MEETING TO MAKE
SURE OUR GOVERNANCE MODELS ARE
EQUITABLE AND HOW DO WE INCLUDE
MORE PATIENT ORGANIZATIONS IN
THAT PROCESS BECAUSE OBVIOUSLY
IN THE SENSE THERE'S A PAY TO
PLAY PARTICIPATION REQUIREMENT
AND PEOPLE WHO SMALL
ORGANIZATIONS REALLY HAVE A
STRUGGLE WITH THAT AND YET THEIR
PERSPECTIVES AND KNOWLEDGE IS SO
RELEVANT TO HOW WE DESIGN THE
PROGRAMS AND HOW WE SUFFICE TO
SAY WE ARE A SMALL ENOUGH
ORGANIZATION THAT WE CAN AFFORD
TO TEST PEOPLE SO WE ARE
REQUIRING PEOPLE TO BE TESTED
EACH WEEK THEY COME TO THE
OFFICE AND LIKE EVERYONE, THIS
IS BEEN A REALLY DIFFICULT TIME
FOR RECRUITING AND RETAINING THE
PROFESSIONALS WE NEED OF HIGH
PREPORTION OF OUR TEAM HAVE
PHDs BUT WE HAVE OTHER PROGRAM
SUPPORT, PROFESSIONALS THAT WE
CAN'T LIVE WITHOUT AND SO WE ARE
BROADENING OUR RECRUITMENT TO A
NATIONAL BASIS AND WE ARE
LEARNING LIKE EVERYONE ELSE TO
WORK REMOTELY AND SO ON AND SO
FOURTH SO THIS IS A LITTLE BIT
OF A PLUG HERE BECAUSE IF YOU
KNOW SOMEONE WHO IS REALLY
INTERESTED TO THIS KIND OF
COLLABORATIVE RESEARCH, ASK THEM
TO CHECK OUT OUR WEBSITE BECAUSE
WE ARE RECRUITING AND WE LOVE TO
THE THE BEST AND BRIGHTEST
TALENT AND THE FINAL SLIDE, THIS
IS IMPORTANT.
I WANTED TO MENTION THAT I SAID
SCIENTISTS AND SCIENCE BUT WE
ALSO HAVE THIS LONG STANDING
COMMITMENT TO SUPPORTING
TRACTOR-TRAILER CARE OF OUR
PATIENTS AND ONE OF THE WAYS
THAT WE'VE DONE THAT IS THROUGH
THE STAFF OR FOUNDATION IS TO
SUPPORT THE FAMILY LODGE ON THE
NIH CAMPUS WHICH IS LIKE THE
CHILDREN'S AND IT'S AN
OPPORTUNITY FOR THE FAMILIES OF
PEOPLE WHO ARE RECEIVING CARE AT
THE NIH CLINICAL CENTERS TO HAVE
A COMFORTABLE AND SUPPORTIVE
ENVIRONMENT DURING THEIR
OTHERWISE DIFFICULT CHALLENGE OF
RECEIVING CARE FOR COMPLICATED
DISEASES.
WE ALSO ANNOUNCED THE ESTATE
GIFT WHICH MENTIONED IS ONE OF
THE WAYS THAT WE SUPPORT
SCIENTISTS BY SUPPORTING Dr.
BONOMAN RESEARCH ON MUSCULAR
DYSTROPHY AND WE WILL BE WORKING
TO INCREASE THIS GIVING GOING
FORWARD.
AND FINALLY ON MY LAST SLIDE, I
JUST MENTIONED MY OWN PRIORITIES
AS ANNAN BOARDING PERSON AND
PART OF THAT IS LISTENING AND
LEARNING AND I HAVE REACHED OUT
TO VARIOUS PATIENT ORGANIZATIONS
TO HAVE THESE KINDS OF
CONVERSATIONS.
I'M INTERESTED TOOULD BE HELPFUR
CURRENTLY BEING HELP AND WHAT
SHOULD WE BE DOING BETTER AND
HOW CAN WE PARTICIPATE IN
PATIENT ORGANIZATIONS NOT JUST
IN THE DC AREA BUT OBVIOUSLY
NATIONALLY AND HOPEFULLY AT SOME
POINT ORGANIZATIONALLY AND WE
ARE REFRESHING OUR STRATEGY AND
I'VE MENTIONED THE WORKPLACE AND
CULTURE ISSUES THAT ARE COMMON
TO US IN THE ORGANIZATIONAL
WORLD AND OUR TEAMS ARE
EVERYTHING AND IF WE DON'T DO
THAT RIGHT WE CAN'T BE
SUCCESSFUL AND IN TERMS OF
SCIENTIFIC PARTNERSHIP
INVESTMENTS OBVIOUSLY WE TEND TO
CONTINUE THE AMPS AND THE
BIOMARKERS CO CONSORTIUM AS WELS
OUR CONTRIBUTION IN THE AREA OF
DIAGNOSTICS AND VACCINES AND
WE'LL CONTINUE AND IT'S NOT IN
THE REAR VIEW MIRROR FOR US SO
WE ARE VERY MUCH CONTINUING TO
BE INVOLVED.
WE JUST HAD THE EXECUTIVE
COMMITTEE MEETING THIS MORNING
WITH THE DIRECTOR OF THE NIH AND
THE COR TEAMS AND WE ARE STILL
WORKING ON THIS PANDEMIC
DILIGENTLY AND FINALLY, I THINK
IN THIS ERA WHERE THE TRUST AND
SCIENCE IS AT AN ALL-TIME LOW,
IT'S REALLY IMPORTANT THAT ALL
OF US, IN THE HEALTH COMMUNITY
STANDS STRONG ON THE PRINCIPLES
OF SCIENCE AND THE IMPORTANCE OF
SCIENCE AND CLINICAL RESEARCH
AND THAT WE KIND OF WORK
TOGETHER TO FIND WAYS TO
EMPHASIZE HOW IMPORTANT ALL OF
THIS IS TO PEOPLE AND TO
PATIENTS EVERYWHERE.
SO I'LL STOP THERE AND ON MY
LAST SLIDE I HAVE MY E-MAIL
ADDRESS SO IF YOU HAVE SOME
IDEAS, COMMENTS CRITICISM OR
COMPLIMENTS, GET IN TOUCH WITH
ME AND I'LL DO MY BEST TO BE
RESPONSIVE.
THANK YOU.
>> THANK YOU SO MUCH Dr.
GERBERDING.
UNFORTUNATELY, Dr. KOROSHETZ
HAD TO RUN FOR ANOTHER MEETING
THAT GOTTA JUSTED BUT HI, NICE
TO MEET YOU, I'M Dr. SAMANTHA
WHITE OUR RUN OUR BRANCH HERE
AND I REALLY ENJOYED YOUR TALK.
I MEAN IT REALLY RESINATES WITH
ME IN THE ENGAGEMENT AND WITH
THE FORUM IN OUR THEME OF
PROGRESS THROUGH PARTNERSHIPS,
WHICH YOU KNOW VERY MUCH ABOUT.
SO, I WANT TO ENCOURAGE EVERYONE
TO POST QUESTIONS IN THE CHAT SO
THAT I CAN HELP Dr. GERBERDING
GO THROUGH A BRIEF Q&A PERIOD.
I SEE ONE WE GOT.
WHAT ELEMENT MAKES YOU THE MOST
EXCITED ABOUT WORK AT THE FNIH
IN THE NEXT SIX TO 12 MONTHS?
>> WOW.
WELL, FIRST, LET ME JUST SAY,
THE REASON I AM GOING TO HAVE A
HARD TIME ANSWERING THIS IT'S
JUST LIKE BEING IN A CANDY STORE
OF SCIENCE.
OF COURSE I'VE BEEN IN THE
SCIENTIFIC ENVIRONMENT MY ENTIRE
CAREER BUT THIS IS CLOSE TO THE
SCIENTISTS AND THE DISCOVER SO
TO SPEAK SO IT'S JUST THRILLING
TO BE BACK IN THAT AND BE
EXPERIENCING THAT.
I THINK IN TERMS OF WHAT CAN WE
ACCOMPLISH IN THE NEXT SIX
MONTHS, I WOULD HAVE TO SAY THAT
THERE ARE FRONTIERS ON THE
BIOMARKERS' HORIZON THAT ARE
EXCITING AND I PROBABLY DON'T
WANT TO SAY SPECIFICALLY WHICH
ONCE I'M TALKING ABOUT BECAUSE
IT MIGHT NOT BE READY FOR PRIME
TIME BUT THERE ARE IMPORTANT
ADVANCES IN THOSE SPACES AND I
THINK OF COURSE, WE HAVE THE ON
GOING WORK AND WHETHER OR NOT
WE'LL GET TO A POINT WHERE WE
HAVE THEIR PIECE FOR LONG COVID
SO STAY TUNED.
>> STAY TUNED.
WE WILL.
SO, ANOTHER QUESTION IS COMING
IN FROM ONE OF OUR EXECUTIVE
COMMITTEE MEETING MEMBERS AND
SHE'S THANKS YOU FOR THE
PRESENTATION AND DO YOU SEE THE
FNIH HELPING TO ENGAGE YOUNG
MINDS IN SCIENCE FOR RESEARCH?
>> YOU KNOW, BECAUSE THE
PIPELINE OF SCIENTISTS IS SO
IMPORTANT TO THE NIH MISSION, IS
CLEARLY IN SCOPE FOR US AND WE
DO SOME SMALL THINGS IN THAT
REGARD BUT WE DON'T HAVE A LARGE
PROGRAM THERE AND THIS IS ONE OF
THOSE AREAS WHERE AS WE WORK ON
OUR STRATEGIES, AND THINK ABOUT
HOW WE GROW THE FOUNDATION,
FINDING PEOPLE WHO ARE
SPECIFICALLY INTERESTED IN THAT
AND WANTING TO SUPPORT STEM
EDUCATION OR JUST BASIC LIT RA
SHE IN SCIENCE I THINK THAT'S A
REALLY IMPORTANT OPPORTUNITY.
ONE OF MY FANTASIES IS TO
PERHAPS TRY TO BE PART OF A
CONVENING OF A COALITION 6 OF
PEOPLE WHO ARE AS INTERESTED IN
THE TRUST ISSUES AS I AM AND
THINK ABOUT HOW KIDS CAN BE PART
OF THAT CONVERSATION BECAUSE IF
WE CAN BRING PEOPLE UP IN A
MANNER FOR THEIR HEALTH LITERACY
FOR THAT MATTER WE'LL HAVE A NEW
GENERATION THAT'S BETTER
EQUIPPED TO PARTICIPATE IN THESE
BROADER SOCIETAL CHALLENGES THAT
WE FACE.
>> YEAH, ABSOLUTELY.
SO ANOTHER QUESTION THAT'S COME
IN FROM LOUIS MAZWAY AND A POLL
GOES IF I MISPRONOUNCE ANYONE'S
NAME.
HE IS LOOKING FOR CLARIFICATION.
WHY WOULD SOMEONE GIFT TO FNIH
AS OPPOSED TO A PARTICULAR
HOSPITAL OR HOSPITAL SYSTEM OR
PARTICULAR NON PROFITS.
CAN YOU HELP THEM UNDERSTAND
KIND OF WHERE FNIH HANGS OUT IN
THE LANDSCAPE?
>> YEAH, I THINK OUR FOCUS IS ON
SCIENCE AND WHERE THAT HAPPENS
AND NOT EVERY SCIENTIFIC PROBLEM
BENEFITS FROM A COLLABORATION OF
MULTI SECTOR COLLABORATION BUT
SOME OF THE HARD PROBLEMS DO AND
I THINK PEOPLE WHO SOPHISTICATED
DONORS SO TO SPEAK, UNDERSTAND
THE PROCESS OF SCIENCE IS
ULTIMATELY AT ITS CORE A
COLLABORATIVE PROCESS.
NO ONE MAKES PROGRESS BY
THEMSELVES AND SO BY
FACILITATING AND BROKERING AN
ORGANIZATION THAT HAS A PRETTY
GOOD TRACK RECORD OF MOVING
THINGS FROM IDEAS THROUGH THE
WHOLE TRANSLATIONAL PROCESS TO
REGULATORY APPROVAL, IS THE WAY
THAT ULTIMATELY MAKE SURE THAT
YOUR INVESTMENTS IS GOING TO
HAVE A HIGH VALUE AT THE END OF
THE DAY AND I THINK THAT'S ONE
OF THE ASPECTS OF OUR ENGAGEMENT
THAT WE NEED TO MAKE MORE
VISIBLE, THIS IS A HIGH POS AND
WE HAVE A REALLY GOOD TRACK
RECORDS OF MOVING THINGS THROUGH
THE PIPELINE.
>> YEAH, ABSOLUTELY.
SO ANOTHER QUESTION HAS COME IN
HOW DO YOU SEE TECHNOLOGIES I
OBJECT INNOVATION AND IN
INITIATIVES AND FOCUS AND
DIGITAL BIOMARKERS.
>> WELL, ABSOLUTELY.
THIS IS A STRATEGIC QUESTION
THAT MY TEAM IS ENGAGING ON AND
SO THAT IS A FARE OF INNOVATION
THAT IS A NO-BRAINER RIGHT NOW
SO WE'LL BE THINKING ABOUT WHERE
THAT MIGHT BE USEFUL AND EVEN IN
MENTAL HEALTH DISORDERS AND
NEWER LOGIC DISORDERS THE
DIGITAL MONITORING IS PATIENT
FRIENDLY AND PROVIDES A UNIQUE
KIND OF INFORMATION THAT
ULTIMATELY SPEAKS TO PATIENT
REPORTED OUTCOMES AS MUCH AS THE
BIOLOGICAL INDICATORS OF SUCCESS
SO I THINK IT'S TIME HAS COME.
>> YES, ABSOLUTELY.
SO ANOTHER QUESTION FROM SARAH,
I'M GOING TO KIND OF PIVOT A
LITTLE BIT IN TERMS OF HOW THIS
IS FRAMED AND YOU KNOW, AS YOU
ARE AWARE ABOUT 10 INSTITUTE AND
HAVE AT NIH THAT A NEUROSCIENCE
FOCUS NINDS IS ONE LEADER AND
WEST NATIONAL INSTITUTE OF
MENTAL HEALTH OR THE NATIONAL
EYE INSTITUTE AND SO ON AND SO
CAN YOU HELP US FIGURE OUT HOW
IT WORKS WITH INDIVIDUAL
NEUROSCIENCE AND INITIATIVES
ACROSS NIH BUT AS WELL A
INITIATIVE LIKE FOR EXAMPLE A
CROSS INSTITUTE INITIATIVE IN
NEUROSCIENCE IS THE BRAIN
INITIATIVE FOR ADVANCING NEURO
TECHNOLOGIES.
>> YEAH, THIS IS PART OF I
THINK, WHERE THE CROSS
DISCIPLINARY APPROACH BECOMES
REALLY IMPORTANT AND OFTEN, THE
AREAS OF COLLABORATION ARE
DRIVEN BY THE NIH DIRECTOR OR A
SUBSET OF INSTITUTES THAT SEE
THE NEED BUT DO ALWAYS HAVE THE
MECHANISMS -- DON'T ALWAYS HAVE
THE MECHANISMS TO CREATE A
COLLABORATION IF YOU THINK ABOUT
PARTICIPATING IN EVERYBODY
THINKS WELL GEE, MAYBE IT WOULD
BE GREAT TO HAVE THESE INDUSTRY
PARTNERS COME INTO PLAY.
YOU CAN'T HAVE EACH INSTITUTE
REACH OUT TO EACH PARTNER TO THE
COORDINATION OF ALL THAT FIRST
AND FOR MOST HAPPENS WITHIN THE
NIH BEFORE WE SEE IT AND THEN
THE GOVERNANCE PROCESS REALLY
PUTS AT THE TABLE THE LEAD
INSTITUTE OR THE LEAD SCIENTISTS
TO BE THE KIND OF PRINCIPLE OF
THE PROCESS BUT WE WOULD MAKE
SURE THAT THE RELEVANT
INSTITUTES ARE INCLUDED ON THE
STEERING COMMITTEES AND
PARTICIPATING IN THE ACTUAL
DESIGN AND CONDUCT OF THE THE
SCIENCE.
THAT'S WHAT I THINK IS REALLY
THE AMAZING THING ABOUT THIS.
COLLABORATIONS HAVE A HIGH
TRANSACTION AT CLASS BUT THEY
PLAN HORIZONTAL LOW AND THEN
EXECUTE VERTICALLY AND I THINK
THAT IS THE SECRET TO THIS KIND
OF PROJECT MANAGEMENT.
ABSOLUTELY.
>> I'M GOING TO ASK YOU A
QUESTION THAT I'M INTERESTED IN
AND ALSO AS I DO, I'M GOING TO
PUT THE FNIH WEBSITE INTO THE
CHAT.
IT'S VERY SIMPLE.
ONE OF THE MY QUESTIONS WAS, I
REALLY APPRECIATED THAT FNIH IS
VERY EXPLICIT OF PUBLIC-PRIVATE
PARTNERSHIP CRITERIA SO I'M
CURIOUS FOR THE DIVERSE
ATTENDEES OF THE FORUM WHO MIGHT
BE ACTUAL LEADERSHIP IN
DIFFERENT SIZE NON-PROFIT
ORGANIZATIONS AND THEY MIGHT BE
MEMBERS OF THE ORGANIZATION AND
MIGHT BE THE PATIENTS AND THEIR
CAREGIVERS WHO ARE SUPPORTED BY
THE ORGANIZATION AND LOOKING TO
THEM.
WHAT ARE THE STEPS THAT THEY
COULD TAKE, IF THEY'RE TRYING TO
UNDERSTAND WHERE THEY FALL IN
KIND OF YOUR PIPE LYNN AT FNIH?
IS THERE A FLOW CHART ON THE
WELCOME BACK SIGHT OR A PERSON
THEY SHOULD E-MAIL?
YOU DON'T WANT ALL OF THESE
E-MAILS GOING TO BE DIRECTLY BUT
REVIEWING THE CRITERIA, IF THERE
ARE GROUPS VERSUS INDIVIDUALS
WHO WANT TO TRY TO ENGAGE IN
THESE PARTNERSHIPS, WHAT WOULD
YOU RECOMMEND THEY DO?
>> SO, RIGHT NOW, THE FIRST
ANSWER IS I DON'T KNOW.
SO THAT IS AN HONEST ANSWER AND
I'M STILL FIGURING THIS OUT BUT
I WILL SAY THAT ONE OF THE FIRST
THINGS I DID WAS TO CREATE A
CONTRACT FOR A PERSON THAT I
WORKED WITH AT MERC WHO LED OUR
PATIENT ENGAGEMENT ORGANIZATION
BUT JANICE RETIRED BUT SHE WILL
CONDUCT AN ASSESSMENT.
FIRST OF ALL, WHAT ARE WE DOING
RIGHT NOW WITH PATIENT
ORGANIZATION THIS IS WHERE ARE
THE PERSPECTIVES AND INSIGHTS
FROM PATIENTS COMING NO THE PLAY
AND WHERE COULD OR THEY SHOULD
AND WHAT IS THE BEST PRACTICE
AND WHAT ARE WE MISSING?
JANUARY WILL MISS OUT TO
ORGANIZATIONS TO GET A POINT OF
VIEW SO WE'LL HAVE CONVERSATIONS
AND INTERVIEWS WITH THOSE FOLKS
AND COMING OUT OF THAT WE'LL
PROVIDE CLARITY AROUND THIS AND
TRUST ME IT'S AN INVITATION AND
I WANT THIS TO HAPPEN?
AND.
>> FANTASTIC.
>> I'M GOING TO ASK YOU ANOTHER
QUESTION BECAUSE IT'S AN AREA
THAT IS IMPORTANT NON PROVE I
CAN PARTNERS AND AND YOU HAD
MENTIONED ON THE STATUS OF FNIH
AND WORKING TOWARDS ONE OF THE
ONES YOU MENTIONED IS EQUITY
DIVERSITY AND INCLUSION AND I
KNOW IN RESEARCH AND WE'RE ALL
WORKING TO MAKE SURE THAT IT
HAPPENS AND THERE'S A SESSION AT
THE FORUM AND HOW TO BE
INCLUSIVE AND SO I'M CURIOUS
LIKE YOU DO GIVE US A FEW MORE
ON HOW GOALS AND AREAS THAT YOU
ARE PAYING FOR IN SIGHT ON FROM
OUR COMMUNITY MEMBERS.
>> I WANT TO START WITH THE AGO
NOMMENT THAT A LOT HAS BEEN DONE
AND SO I DON'T MEAN TO IMPLY
THAT SOMETHING WAS WRONG AND
RAISING INTO FIX IT AND FROM A
GRASSROOTS LEVEL BECAUSE OF THE
IMPOSED ISOLATION OF THE
PANDEMIC AND HALF OF OUR
ORGANIZATION GOT TOGETHER SO I
THINK THAT IT CAN GO PIR IN THE
SPACE AND SOME OF THE AREAS
WHERE WE NEED TO IMPROVE JUST
DON'T THE DIVERSITY OF WORKFORCE
AND THE INCLUSION AND COMFORT
THAT WE CREATE FOR THE BROAD
SPECTRUM OF EMPLOYEES THAT WE
HAVE SO ONE OF THE WORKGROUPS IS
SPECIFICALLY LOOKING AT OUR WORK
AND NOT JUST WHO WE ARE AND HOW
WE INTERACT WITH EACH OTHER BUT
DOES OUR COMMITMENT TO SCIENCE
AND THE MISSION OF THE NIH BUT
ULTIMATELY ARE WE INCLUSIVE IN
THAT SENSE?
THAT'S A HARDER QUESTION.
I DON'T KNOW THE ANSWER BECAUSE
WE HAVE COMPLEX SET OF PROGRAMS
AND THAT IS WHAT I TEND TO FIND
OUT.
AND WE KNOW THE HEALTH
DISPARITIES ARE SO PARAMOUNT AND
WHAT DO WE GET TOGETHER WITH OUR
PARTNERS AND HOW DO WE SELECT
PARTNERS THAT ARE GOOD AT THIS
SO THERE'S AN EXPECTATION THAT
IT'S NOT JUST THAT THEIR THIS
BUT EVERYBODY THAT COMES INTO
KIND OF THIS PART AND IT'S
COMMITTED AND THAT IS WHAT IS SO
GREAT ABOUT THE RECENT MEETING
AND SO WE ARE ADDRESSING THAT
THEY TOOK THE ENTIRE MEETING TO
FOCUS ON THIS ISSUE.
>> IT'S FANTASTIC AND I LOVE
THAT POINT ABOUT IT IS
COLLABORATIVE AND IT'S NOT JUST
ALL FNIH OR ALL NIH IT'S THE
WHOLE COMMUNITY AND TAKING
EXPERTISE SO WE CAN MOVE FORWARD
IN A POSITIVE WAY.
WE HAVE IS LOOKING ANOTHER THE
FUNDRAISING PARTICULARS SO IN
TERMS OF YOU HAD MENTIONED FNIH
AND JUST A LITTLE BIT MORE TWO
THINGS MEAN AND WHAT DOES IT NOT
VERSED IN KIND OF THE WORLD OF
DONATIONS AND HOW GET ARTICULATE
IN THE SYSTEM.
>> EARLY DAYS FOR ME IN THIS
REGARD AND ONE IS THE
TRADITIONAL FILL AN THROW I CAN
APPROACH GOING WAY BACK 25 YEARS
AGO AND THE PEOPLE WHO WERE PART
OF THE ORIGINAL BOARD OF THE
FOUNDATION HAD STRONG
PHILANTHROPIC TENDENCIES AND WE
HAVE SUPPORT THAT WAS
LONGSTANDING AND WE ALSO HAVE
SPECIALEN COUPLES AND AND THEY
SUPPORT IN WAYS AND THE I
SKIPPED OVER THE SLIDE AND I
DESCRIBED OUR ANNUAL AWARDS
DINNER AND WE NOMINATE
PARTICULARLY TALENTED AND
PRODUCTIVE SCIENCES AND AN AWARD
AND THAT IS SOMETHING THAT HAS
BEEN SUPPORTED BY FOR A NUMBER
OF YEARS OR GRATEFUL AND IT'S 10
YEARS AND SO THAT IS THE KIND OF
THING THAT WE TRY TO BUILD ON
GOING FORWARD AND WE HAVE A
DIFFERENT FUNDRAISING AND THAT'S
PROJECT FUNDRAISING AND THE
FOLKS THAT ARE INTERESTED IN ARE
PROJECT AND THOSE WHO ARE
STAKEHOLDERS AND INCLUDES
INDUSTRIES AND BIOTECH OR
PARTNERS FOR THE MOR DIGITAL
APPROACH TO WHAT WE'RE DOING AND
IN ADDITION TO THIS WE DO
RECEIVE SIGNIFICANT RESOURCE 0
FOUNDATIONS AND I MENTIONED
GATES BUT THERE ARE OTHER
FOUNDATIONS WHO HAVE A SPECIAL
INTEREST IN A PROJECT AREA OR
JUST THE ADVANCEMENTS AND THE
HARTEST THINGS FOR US TO
ACCELERATE THE START UP OF SOME
OF OUR FUNDS SO NOT HAVING TO
WAIT UNTIL UNTIL THE MONEY WAS
THE SPTO --WE'VE LEARNED THAT AO
NOW WE HAVE TO FIGURE OUT HOW
CAN WE ARE PART OF THAT ENGINE
AND IT DRIVES THE PROGRESS
FORWARD AND MORE EXPEDITION US
MANNER.
>> FANTASTIC.
SO WE DID JUST RECEIVE ONE LAST
QUESTION THAT WE DO HAVE A
COUPLE MINUTES FOR BUT I WANT TO
JUST ACKNOWLEDGE AGAIN THAT YOU
HAVE RECENTLY TAKE NOT ON THIS
FANTASTIC ROLE SO IT'S
CONCEIVABLE YOU KNOW THE ANSWER
TO THIS BUT THAT'S OK WE CAN PUT
A PIN IN IT IF NOT.
ONE OF OUR GROUP WAS SO
DELIGHTED BY YOUR TALK BECAUSE
THEY DIDN'T EVEN KNOW NIH
EXISTED SO THIS WAS FANTASTIC
AND WHAT WE WERE TRYING TO DO.
THE QUESTION IS, KIND OF, ABOUT
MORE OF A TARGETED RESEARCH
PROJECT IN THE NEUROSCIENCES AND
SO YOU MENTIONED ONE OR TWO AND
IS THERE ANYTHING THAT FNIH HAS
ITS FINGERS IN WITH WITH REGARD
TO BRYN INJURY AND THE AND OF
THAT AND I KNOW THAT THE HISTORY
AND THE PROCESS AGENCIES AND I
HAVE TO CHECK ON THAT WOULD BE
SURE AND I DO JUST REALLY WANT
TO SAY IN CLOSING THAT I
APOLOGIES FOR NOT BEING FULLY
INFORMED ABOUT EVERYTHING, IT'S
JUST BEEN DRINKING FROM A FIRE
HORSE AND I WOULD ALSO JUST SAY
WHEN YOU THINK ABOUT FNIH AND
THINK ABOUT IT IN THE SENSE OF
THE MOUSE THAT ROARED AND WE
WANT TO STAY IN THE GOLDILOCKS
STAGE OF BEING VISIBLE ENOUGH TO
DO OUR WORK WELL AND BE HELPFUL
TO THE NIH AND OUR PATIENTS BUT
AT THE SAME TIME, WE'RE NOT GET
BIG AND GO HIGHWAYLY VISIBLE AND
FRONT-RUNNER HERE AND WE'RE VERY
HAPPY TO BE CATALYST OF GREAT
SCIENCE AND ULTIMATE LOW TO DO
WHAT I HOPE WILL BE BETTER
HEALTH FOR THE PEOPLE THAT
REALLY FEET N AND HOPEFULLY I'LE
MORE INFORMED.
>> YOU DID AN AMAZING JOB AND
THANK YOU FOR GIVING US YOUR
TIME.
I KNOW IT'S BEEN DRINKING FROM
THE FIRE HORSE AND I APPRECIATE
ALL THE INFORMATION YOU SHARED
AND WE ARE GOING TO ARCHIVE THIS
SO THAT THOSE WHO WANT TO
REFERENCE IT AGAIN IN THE NEWT
ARE ABLE TO THANK YOU SO MUCH
AND HAVE A WONDERFUL DAY AND I
HOPE YOU ARE ABLE TO JIN OUR
SESSIONS IN THE NEXT TWO DAYS.
>> ENJOY THE REST OF THE
MEETING, EVERYONE.
THANK YOU.
>> THANK YOU.
>> SO WITH THAT, I'M GOING TO
SEG WAY US INTO OUR NEXT SESSION
AND THIS IS A SESSION THAT IS
GOING TO BE LED BY Dr. GERALD
DEAN ACUNA SUNSHINE AND PATIENT
ADD VOW SEE IN COMMERCIALIZATION
HOW TO WORK WITH INDUSTRY SO I
JUST WANT TO GIVE A FEW REMARKS
ABOUT GERALDINE SHE'S THE
PRESIDENT OF THE SUNSHINE CARE
FOUNDATION FOR NEUROLOGICAL CARE
AND RESEARCH AND A CO-FOUNDER OF
THE CENTER FOR DIS TONE YAR
PARKINSON'S AT THE MASSACHUSETTS
GENERAL HOSPITAL.
THESE ARE TWO INTERNATIONAL
NON-PROFIT ORGANIZATIONS, TWO,
DEDICATING TO FINDING
NEUROSCIENCE RESEARCH AND
FINDING WAYS OF DELIVERING FRO
CLINICAL CARE AND HUMANITARIAN
AID TO INDIGENOUS ENTER PATIENTS
IN RURAL AREAS OF AISH AMOUNT
SHA.SHE WAS THE FIRST BRAIN BANN
THE PHILIPPINES NOW PRIOR, SHE
ENTERED NO THE NON-PROFIT AND
SOCIAL IMPACT SECTORS, SHE
PRACTICED CORPORATE LAW FULL
TIME AS SENIOR COUNCIL IN
ALTERNATIVE INVESTMENTS AT BRACE
BRIDGE CAPITAL IN BOSTON AND
SERVED ON THE HARVARD UNIVERSITY
OF OVERSEERS AND THE PRESIDENT
COUNCIL OF MGH AND SHE SITS ON
THE VISITING COMMITTEE FOR THE
HARVARD SCHOOL OF PUBLIC-HEALTH
AND THE HARD SAD INITIATIVE AND
SHE LIVES IN BOSTON AND AS WELL
AS MANILA IN THE PHILIPPINES.
I'LL TAKE IT AWAY AND I DO WANT
TO NOTE, I'M SO SORRY OTHER, ONE
LAST THING, THAT I WOULD LIKE TO
ENCOURAGE OUR REGISTRANTS TO DO.
WE'RE GOING TO PASTE IN THE CHAT
THE OPPORTUNITY TO CONTINUE TO
SCHEDULE ONE-ON-ONE MEETINGS
WITH NINDS PROGRAM STAFF OVER
THE NEXT TWO DAYS AND ON
WEDNESDAY AND I ALSO WANT TO
ENCOURAGE ALL OF OUR NON NIH
PARTICIPANTS TO CONSIDER FILLING
OUT A SURVEY THAT WAS CREATED BY
THE NON-PROFIT MEMBERS OF OUR
EXECUTIVE BOARD FOR A BREAKOUT
SESSION THAT IS AT THE END OF
DAY TWO.
APOLOGIES.
THE FLOOR IS YOURS --
>> AWESOME.
THANK YOU SO MUCH, SAM.
GOOD MORNING, EVERYONE, WELCOME
TO OUR FIRST PANEL DISCUSSION OF
THE MORNING AND THANK YOU TO THE
ENTIRE NINDS TEAM FOR THIS
AMAZING OPPORTUNITY FOR US TO
GATHER IN THIS FORUM, AS WAS
MENTIONED MODERATOR FOR THIS AND
AND COMMERCIALIZATION AND HAVE
TO WORK WITH INDUSTRY AND AS
MENTIONED I CO FOUNDED THE
CENTER FOR DIS TONIA AND IT'S A
RARE ADULT ONSET
NEURODEGENERATIVE DISEASE FOUND
IN PATIENTS WITH ANCESTRY FROM
THE PHILIPPINES.
SO, WE HAVE AN AMAZING GROUP OF
PANELISTS TODAY.
BUT BEFORE WE BEGIN, THEY DID
WANT ME TO CLARIFY BECAUSE OF
THE SESSION TITLE, THAT OUR
DISCUSSION WILL NOT BE FOCUSED
ON THE COMMERCIALIZATION OF
THERAPEUTICS, PER SE, BUT RATHER
ON HOW PATIENT ADVOCACY GROUPS
AND COMMERCIAL ENTITIES CAN WORK
TOGETHER TO FIND THERAPEUTICS
SOLUTIONS FOR THEIR RELEVANT
DISEASES.
AFTER THE PANELIST DISCUSSION,
WE WILL HAVE 10 TO 15 MINUTES IN
THE END FOR YOUR QUESTIONS AND
IF YOU WITH PUT THEM IN THE
CHAT, YOU WILL CALL ON YOU AT
THE END OF THE MEETING.
SO, JOINING US SO JOINING
ME IS CATHERINE BEAVERSON, AND
CONNECTS PATIENTS AND INDUSTRY
PARTNERS AND FOCUSED ON
ULTRARARE NEURO DEVELOPMENTAL
DISORDERS IN THEIR JOURNEY TO
DRUG DEVELOPMENT AND LAURA MITIC
CHIEF SCIENTIFIC OFFICER AT
BLUEFIELD PROJECT TO CURE FTD.
AND I BELIEVE WE ARE ALL THERE,
WE HAVE NO SLIDES SO HERE WE
ARE.
ALL OF US ON THE SCREEN.
SO, WELCOME, THANK YOU AND LET'S
START WITH CATHERINE, WELCOME,
GOOD MORNING.
>> GOOD MORNING.
>> YOU KNOW, AS WE DISCUSSED
DURING ONE OF OUR THREE
CONFERENCE CALLS, I COME ACROSS
A 2015 STUDY CONDUCTED BY SOME
RESEARCHERS AT DUKE ON THE
HURDLES TO COLLABORATION AMONG
PATIENT GROUPS, INDUSTRY
PARTNERS AND AK DEEM I CAN AND
WHILE INDUSTRY IDENTIFIED
INTERNAL BUREAUCRACY AS ITS
GREATEST HURDLE TO
COLLABORATION, PATIENT GROUPS
FOCUSED ON THE LACK OF
TRANSPARENCY AND UNDERSTANDING
OF HOW TO ENGAGE WITH INDUSTRY
AS THEIR GREATEST HURDLE.
AND IN THAT DISCUSSION, YOU
SHARED WITH ME A LINK TO
FRAMEWORK THAT I FOUND REALLY
HELPFUL DEVELOPED BY THE
CLINICAL TRIALS TRANSFORMATION
INITIATIVE.
IT'S A GROUP OF ACADEMICS,
PEOPLE AT THE FDA, PATIENT
ADVOCATES AND INDUSTRY INCLUDING
PEOPLE AT PFIZER, THAT DEVELOPED
THIS FRAMEWORK WHICH I FOUND
REALLY HELPFUL.
AND I BELIEVE THAT WE HAVE THIS
LINK IN THE CHAT AND I WONDERED
IF YOU COULD LEAD US THROUGH AND
GIVE A BRIEF OVERVIEW OF THE
CTTI FRAMEWORK BECAUSE I FOUND
IT VERY HELPFUL.
>> THANK YOU IT'S A GREAT
QUESTION AND I'M HAPPY IT TALK
ABOUT THIS FRAMEWORK AND THE
MODEL THAT CTTI HAS PUT TOGETHER
AND LET'S QUALIFY THAT WITH
THERE ARE A SHARED PURPOSE IN
HOW DO WE ENHANCE THE QUALITIES
AND THE EFFICIENCY OF THE
CLINICAL TRIAL PROCESS AND
EXPERIENCE.
THE STUDY THAT YOU REFERENCED
REALLY SAID OK, WELL, WHO ARE
THE STAKEHOLDERS INVOLVED IN
THIS PROCESS AND WHO DO SHARE IN
THIS PURPOSE AND THIS MISSION AS
YOU MENTIONED, ACADEMICS,
SPONGESSORS OF CLINICAL RESEARCH
AND PATIENT COMMUNITIES HAVE A
SHARED INTEREST BUT YOU HAVE TO
START FROM PLACE OF WELL, YOU
KNOW, HOW WELL DO WE KNOW EACH
OTHER AND HOW WELL DO WE KNOW
OUR CAPABILITIES AND HOW WELL DO
WE KNOW OUR OWN CAPABILITIES AND
THAT INITIAL STUDY WRAPPED OUT
WHERE ENTITIES SAW THE
CHALLENGES TO ENGAGING WITH EACH
OTHER BUT THEIR PERCEIVED VALUE
NAD THAT DISCUSSION AND COMING
OUT OF THAT WORK THIS MODEL THAT
FOLKS HAVE BEEN ABLE TO OPEN UP
AND TAKE A LOOK AT WAS REALLY
PUT FORWARD TO SAY HERE THE
OPPORTUNITIES AND PATIENT GROUPS
CAN TAKE TO ENGAGE IN THE
CLINICAL TRIAL CONTINUUM
RECOGNIZING WHAT ARE SOME OF THE
SCIENTIFIC MILESTONES THAT NEED
TO BE ACHIEVED IN ORDER TO MOVE
FORWARD IN THE PROCESS AND SO
THE MODEL NICE LOW LAYS OUT AT A
HIGH LEVEL, THAT THIS STAGE OF
DEVELOPMENT LOOKING AT KIND OF
DISCOVER RODISCOVERY AND PRE CLK
AND THE REASON TO BELIEVE FROM A
SCIENTIFIC RATIONALE STANDPOINT
AND MOVING INTO THOSE CLINICAL
PHASES OF DEMONSTRATING SAFETY
AND EFFICACY AND WHAT DOES A
REGULATORY REVIEW LOOK LIKE AND
THE DECISION MAKERS AND THE DATA
PACKAGE THAT IS SUBMIT AND WHAT
NEEDS TO HAPPEN IN A POST
APPROVAL PROCESS.
SO NICELY LAYS OUT WHAT ARE
THOSE MAJOR MILESTONES THAT NEED
TO BE ACHIEVED ALONG THE
DEVELOPMENT PATHWAY AND WITHIN
THOSE STAGES WHAT RECOGNIZES TO
MOVE IT FORWARD AND SO, IN THAT
DISCOVERY AND PRE CLINICAL
SPACE, IT'S REALLY AROUND WHAT
ARE THE STUNTS TO DERISK THE
SCIENCE THAT ENABLES THE
ADVANCEMENT AND TO CLINICAL
RESEARCH AND SOME OF THE
OPPORTUNITIES THERE ARE GRANT
FUNDING OF RESEARCH AND
IDENTIFYING WHAT ARE SOME OF THE
SCIENTIFIC NETWORKS, WHERE THE
MOUSE MODELS AND WHERE ARE THE
AS SAYS BEING DEVELOPED AND ALL
THOSE KINDS OF THINGS AND WHEN
YOU GO INTO OPERATIONALIZING
CLINICAL RESEARCH, HOW CAN
PATIENT GROUPS BE INFORMING
INDUSTRY TO OPTIMIZE THE
FEASIBILITY AND THOSE
OPERATIONAL ASPECTS OF THE TRIAL
WITH A SHARED PURPOSE OF
REDUCING BURDEN ON THE PATIENT
COMMUNITY, RIGHT.
ENHANCING RECRUITMENT AND
RETENTION.
ALL OF THESE THINGS IMPORTANT TO
BOTH PARTICIPANTS AND TO
SPONSORS AND HOW DO WE LEVERAGE
THE EXPERTISE THESE ENTITIES
BRING TO THE TABLE AND FURTHER
ALONG IN THE PROCESS AND HOW DO
PATIENTS ENGAGE IN THAT
DECISION-MAKING AND I THINK WHY
IS THIS THAT PEOPLE CAN COME
TOGETHER AND SPEAK TO EACH OTHER
WHAT IS IMPORTANT FROM A PATIENT
ADVOCACY ORGANIZATION STAND
POINT AND WE MIGHT NOT BE ABLE
TO DO EVERYTHING BUT WE CAN
CHECK AND WE CAN SET OUR
MISSIONS AND OUR CAPE A LOTS
AGAINST WHAT IS NEEDED FOR OUR
COMMUNITY AND HOW WE K WE FIT
INTO THIS FRAMEWORK SO A LONG
WINDED ANSWER, I HOPE THAT WAS
HELPFUL.
>> YES, AND I THINK IT'S GREAT
AND AS STATED IN THE CHAT,
PEOPLE WILL SEE THAT IF THERE'S
MORE ON THE WEBSITE AND I WOULD
JUST ENCOURAGE EVERYONE TO GO ON
THERE AND FIND WHAT MIGHT BE
USEFUL FOR YOU.
SO, LET'S MOVE ON TO YAEL.
YOU'VE SEEN THIS FRAMEWORK AND
THERE'S A EUROPEAN FRAMEWORK
SIMILAR THAT CAN HELP GUY
PATIENTS THROUGH AND MAHZI
THERAPEUTICS AND YOU HAVE A
GROUP OF PEOPLE THAT RUN A
BOOTCAMP THAT GUIDES PATIENTS
THROUGH THIS PROCESS, RIGHT, TO
INTRODUCE AND BRING PATIENT
GROUPS AND INDUSTRY TOGETHER.
CAN YOU TALK ABOUT THAT AND OF
COURSE AFTER THAT, WHAT YOU DO
WITH MAHZI.
>> SURE.
THANK YOU FOR HAVING ME.
SO, I'D LICK TO FOLLOW-UP ON
WHAT CATHERINE WAS DISCUSSING,
THE FRAMEWORK.
I THINK THE FRAMEWORK IS
EXCELLENT AND IT'S A GREAT
STARTLING POINT.
WHAT WE REALIZED DURING MY TIME
AT ULTRA-GENICS AND OF COURSE
NOW WITH MAHZI, THERE ARE A LOT
OF NEW FOUNDATIONS THAT ARE
COMING TO LIFE IN THE LAST FEW
YEARS AND SINCE THAT PUBLICATION
IN 2015, THE LANDSCAPE HAS
CHANGED SIGNIFICANTLY AND THERE
ARE A LOT OF ESTABLISHED
FOUNDATION THAT'S HAVE BEEN
THROUGH INTERACTIONS WITH
INDUSTRY AND THEY HAVE TAKEN
DRUGS AND DEVELOPED THEM AND
HANDED THEM OVER TO PARTNERS BUT
THERE ARE MANY, MANY NEW
FOUNDATION AND SOME OF THEM I
SEE HERE IN THE AUDIENCE AND ARE
REALLY STARTING OUT AND MANY OF
THEM ARE NOT BIOLOGISTS BY
EDUCATION OR DRUG DEVELOPMENT BY
PROFESSION AND THE GAP BETWEEN
THE FRAMEWORK AND ACTUALLY
PUTTING IT TO PRACTICE IS PRETTY
SIGNIFICANT BECAUSE YOU LOOK AT
IT AND NOT UNDERSTAND THE STEPS
BECAUSE YOU'VE NEVER BEEN
THROUGH IT BEFORE SO AFTER
RESPONDING TO MANY INDIVIDUAL
FOUNDATIONS AND PARENTS AND
PATIENTS ON ALMOST A WEEKLY
BASIS AT ULTRA-GENICS, WE
DECIDED TO PUT TOGETHER AN EVENT
THAT WOULD EDUCATE PEOPLE THAT
ARE NEW TO THE DRUG DEVELOPMENT
SCENE OR AREA AND EDUCATE THEM
ABOUT WHAT IT'S ALL ABOUT SO WE
ORGANIZED A THREE-DAY EVENT
WHERE PEOPLE COME TO THE
ULTRA-AGAIN -- WE TRYTO COMPLAT
STARTING FROM HOW TO
COLLABORATION WITH ACADEMICS AND
NEGOTIATE A LICENSE AGREEMENT
ALL THE WAY THROUGH REGULATORY
CLINICAL END POINTS TALKING
ABOUT REGISTRIES AND NATURAL
HISTORY STUDIES FOR THOSE WHO
ARE NEW TO THIS OR MAY BE
STARTED OUT AND KNOW WHAT IS
COMING ON THE HORIZON IT'S A
GREAT OPPORTUNITY TO NOT ONLY
LEARN BUT ALSO INTERACT WITH
EXPERTS AND GIVE TALKS WITH THE
BOT CAMP AND PEERS AND SHARE
BEST PRACTICES.
WE KEEP REFINING AND IMPROVING
THE PROGRAM BASED ON FEEDBACK
FROM PARTICIPANTS AND HOPEFULLY
THEY'LL OPEN A FOLLOW-UP FOR
PEOPLE THOUGH WANT TO KEEP
ENGAGING WITH THE GROUPS THAT
PARTICIPATE TO HELP THEM
THROUGHOUT THEIR JOURNEY ON A
SPECIFIC HURDLES THAT THEY COME
UP WITH AND I SEE HERE IN THE
AUDIENCE THAT THERE ARE A LOT OF
FOUNDATIONS AND DIFFERENT LEVELS
OF MATURITY AND WE DO CATER TO
THE ONES THAT ARE NEWER AND LESS
EXPERIENCED BUT WHEN ANYONE
KNOCKS ON OUR DOOR AND HAS A
SPECIFIC PROBLEM OR ISSUE THEY
NEED HELP WITH WE ENGAGE WITH
THE DIFFERENT EXPERTS AND THE
SPONSORS OF THE BOOTCAMP THAT
LEND THEIR EXPERTISE AND WE TROY
TO HELP ON A CASE-BY-CASE BASIS
AS WELL.
THIS IS GIVEN US ACCESS AND
BETTER UNDERSTANDING OF WHAT THE
ISSUES THAT IS THE FOUNDATIONS
HAVE TO DEAL WITH WHEN THEY'RE
STARTING OUT AND IT COULD BE
LOOKING AT A CHINESE PICTURE
BOOK FOR A NON CHINESE SPEAKER.
WE HAVE OUR OWN TERM ON OLE
GOUGH AND WE TERMINOLOGY SOWE'RS
POSSIBLE.
TO SIMPLIFY THE PROCESS SO
PEOPLE WILL FEEL MORE
COMFORTABLE AS THEY DIVE INTO
DRUG DEVELOPMENT.
THAT'S THE BOOTCAMP.
POST COVID WE'RE REFORM ATING
BUT WE HOPE TO RUN IT TWICE AS
YEAR AND WE DO IT WITH THE
SUPPORT OF GLOBAL GENES AND THE
REGISTRATION GOES TO THEM SO IF
ANYONE WANTS MORE DETAILS,
CONTACT ME AN I'LL CONTACT.
MAHZI I AM INNATED FROM THE
BOOTCAMP BECAUSE I SAW GROUPS
THAT ARE FUNDING BASIC RESEARCH
AND LAURA CAN TALK A LITTLE BIT
MORE ABOUT HOW BLUE FIELD
PROJECT IS HANDLING IT AND A LOT
OF GROW GROUPS ARE FUNDING IT AD
TAKING THE -- THEY DON'T HAVE
THE KNOW HOW OR EXPERTISE TO
TAKE IT FURTHER OR THE FUNDING
INFORM TAKE IT FURTHER BECAUSE A
LOT OF THESE ARE GENETIC BASED
THERAPIES AND THESE ARE NOT
CHEAP AND WE NEED RESOURCES IN
ORDER TO TAKE THOSE INTO THE
CLINIC AND FURTHER AND I STARTED
THE COMPANY TO TRY AND FIRST OF
ALL BE A SOURCE OF INFORMATION
TO GROUPS THAT WANT TO DO THIS
AND CAN COME TO US AND SEEK OUR
ASSISTANCE AND PUTTING TOGETHER
A RESEARCH PLAN AND ALSO WE TOOK
ON THREE PROGRAMS TO GENE
THERAPY AND ONE FOR SPECIFIC
NEURO DEVELOPMENTAL DISORDERS
AND HOPING TO BRING ON MORE AS
TIME GOES BY AND HOPEFULLY
FUNDING WILL COME TO ENABLE US
TO TAKE ON MORE PROGRAMS AND
REALLY WORK HAND AND HAND WITH
THE PATIENT GROUPS WHO BRING
THEIR OWN EXPERTISE, THE
OUTREACH TO THE COMMUNITY AND
THE DEEP UNDERSTANDING OF THE
DISEASE AND THE DISORDER AND
ALSO THE ABILITY TO COLLECT
INFORMATION FROM THE COMMUNITY
AND HELP ENROLL THESE AND BRING
OUR EXPERTISE AND DRUG
DEVELOPMENT AND HOPEFULLY
TOGETHER TAKE A DRUG INTO THE
CLINIC AND MAYBE EVEN
COMMERCIALIZE AND IN THE CHAT,
YOU ARE CONTACT INFORMATION OR
WHERE PEOPLE --
>> I'M HAPPY TO DO THAT.
THAT'S A GREAT SEG WAY TO LAURA
BECAUSE YOUR CHIEF SCIENTIFIC
OFFICER OF BLUEFIELD FOUNDATION
FOCUS OZ ON FRONTING A CURE FOR
FTD AND YOU HAVE WORKED WITH
INDUSTRY AND NOW I HAVE TWO
DRUGS IN CLINICAL TRIALS AND
YOU'VE ALSO SEEN THIS FRAMEWORK
AND HOW DID YOU GUYS START DOING
YOUR WORK WITHOUT THIS KIND OF
ROADMAP AT THAT TIME?
CAN YOU SHARE WITH US A LITTLE
BIT ABOUT YOUR EXPERIENCE?
>> SURE.
THANK YOU.
YES, SO, THE BLUEFIELD PROJECT
WAS FOUNDED IN 2009 AND WE HAVE
A PRE DETERMINED SUNSET DATE OF
2025.
WE'RE NEARING THE END OF OUR
EXISTENCE AND ACTUALLY THERE ARE
FOUR COMPANIES HAVE DRUGS IN
CLINICAL TRIALS FOR THE DISEASE
WE WORK ON WHICH IS FTD CAUSED
BY MUTATIONS IN AN ADULT ONSET
NEURODEGENERATIVE DISEASE
STRIKING IN THE MID-60S WITH A
SIX TO 10-YEAR TIME UNTIL DEATH.
SO, WE ARE VERY FORTUNATE TO
HAVE THE POSITION WHEN WE DO
SUNSET, WE HOPEFULLY WILL HAVE
AN APPROVED DRUGS OR DRUGS IN
TRIALS AND WILL BE APPROVED OR
AT LEAST HAVE READOUTS SOON AND
I THINK THAT WHEN I LOOK BACK
OVER THIS, EACH FRAMEWORK, I WAS
PLEASED TO SEE THAT
INDEPENDENTLY BLUE FIELD HAD
SINCE 2009, REALLY DONE MANY OF
THE THINGS ON THAT FRAMEWORK BUT
I THINK IF YOU ASKED ME IN 2009
ABOUT THAT FRAMEWORK I WOULD NOT
HAVE MANY TERMS ON IT EXPLAINED
TO ME AND SO I THINK THAT BRINGS
ME TO THE POINT IN MANY
FOUNDATIONS THAT ARE ATTENDING
TODAY, IF YOU HAVE NOT ALREADY
AND PROBABLY MANY OF YOU HAVE
AND WHICH BENEFITED FROM THE
EXPERTISE OF OTHER COLLEAGUES AT
OTHER FOUNDATIONS WHO WERE
GENEROUS IN SHARING ALL KINDS OF
INFORMATION AND EVEN A TEMPLATES
FOR FUNDING AGREEMENTS AND ALL
KINDS OF THINGS AS WE STARTED ON
AND IN VARIOUS STAGES ALONG THE
ROAD AND SO I WOULD ENCOURAGE
ALL OF US TO JUST CONTINUE TO BE
AS COLLEGIAL AND COLLABORATIVE
AS WE ARE IN SHARING OUR
INFORMATION AND OUR EXPERIENCES
AND ALONG THIS WAY BECAUSE
THERE'S NO REASON TO REINVENT
THE WHEEL AND I THINK MANY OF
THE ITEMS ON THIS CTI FRAMEWORK
CAN BE CONDUCTED SIMULTANEOUSLY
AND BECAUSE TIME IS ALWAYS OF
THE MOST URGENCY IN THESE
DISEASES AND BEING ABLE AND
SMALL FOUNDATION TO DO THINGS
SIMULTANEOUSLY REALLY MEANS
PIGGING BACKING ON THE EFFORTS
OF OTHERS.
I THINK THAT THE ROADMAP, WE'RE
AN AT THE FOURTH PART OF THAT
ROADMAP AND I HOPE WE WILL BE
AND I HOPE I CAN REFER TO IT FOR
GUIDANCE AS WE HAVE OUR
FORTUNATE ENOUGH TO BE IN THAT
POST APPROVAL SITUATION BUT I DO
THINK THE FRAMEWORK IS HELPFUL
AND A GO AHEAD THING TO REFER
BACK TO OVER TIME.
>> THAT'S AWESOME.
CATHERINE, YOU WANTED TO ADD
ABOUT THE TOOLS.
THIS IS ALL ABOUT TOOLS AND WHAT
WE CAN USE TO HELP OUR -- I JUST
WANT TO BUILD ON THE COMMENTS IN
THAT THIS IS A HIGH-LEVEL
FRAMEWORK THAT IS DIFFICULT TO
TRANSLATE TO THE TOOLS THAT WE
NEED TO ACTUALLY HAVE IT AND THE
BOOTCAMP IS JUST A GREAT
OFFERING AND FOR FOLKS
UNDERSTANDING THIS AND THERE ARE
NEW COMPANIES ALWAYS COMING INTO
THE RARE DISEASE SPACE, THAT
NEED INTERNALLY, TO BE ABLE TO
HAVE TOOLS FOR SYSTEMATIC
EASTBOUND GAGEMENT RIGHT THAT IS
WHAT THE PATIENT COMMUNITIES
WANTS AND HOW THEIR COMPANY
FEELS THEIR FREE TO OPERATE AND
I KNOW PFIZER HAS TAKEN GREAT
EFFORT TO BUILD INTERNAL TOOLS
FOR COLLEAGUES INVOLVED IN
CLINICAL RESEARCH TO DO THIS
APPROPRIATELY, SYSTEMATICALLY
AND CONSISTENTLY AND IN A WAY
THAT IS PALLETABLE BY THE
PATIENT COMMUNITY.
SO I JUST WANTED TO ADD THAT
INTERNAL TOOLS FOR COMPANIES IS
JUST AS IMPORTANT AS BEING ABLE
TO SHARE AND DEVELOP TOOLS
EXTERNAL LOLY FOR FOLKS.
>> CATHERINE, THERE ARE PEOPLE
IN ATTENDANCE WHO ARE JUST
BEGINNING THEIR JOURNEYS HERE
AND ALL OF THIS IS TRYING TO
FIND THE RIGHT PARTNER, RIGHT,
TO ACHIEVE OUR GOALS.
SO, WHAT WOULD YOU SAY PATIENT
ADVOCATE GROUPS WHO ARE STARTING
OUT OR TRYING TO FIND THEIR WAY
SHOULD MAKE SURE THEY DO?
TO TRY TO FIND THE RIGHT PARTNER
AND WHAT DOES THE RIGHT FIT LOCK
LIKE?
FROM YOUR PERSPECTIVE?
>> YEAH, I DON'T KNOW I'M AN
EXPERT ON THE RIGHT FIT.
YOU KNOW, IN TERM OF WHAT
ORGANIZATIONS PATIENT
COMMUNITIES SHOULD BE LOOKING
FOR IN THE INDUCE THROW PARTNER
OF CHOICE OR MULTIPLE PARTNERS,
RIGHT, IS A, DO THEY REALLY
UNDERSTAND THE FOCUS OF THE
COMPANY?
IS IT BROAD TO RARE DISEASES?
IS IT SPECIFIC TO SERVING
THERAPEUTIC AREAS?
DO YOU REALLY UNDERSTAND, IS IT
EVEN SPECIFIC TO A PLATFORM OF
OUR PLATFORM TECHNOLOGY AND SO
DO YOU UNDERSTAND WHAT THE
COMPANY IS TRYING TO ACCOMPLISH
WITH REGARD TO ITS DISEASE AREAS
OF FOCUS AND IT'S OWN
CAPABILITIES AND MAYBE
UNDERSTANDING WHAT IS THE LIFE
CYCLE PROCESS?
IS IT TO ENABLE PRE CLINICAL
DEVELOPMENT AND BASIC RESEARCH
OR IS IT REALLY TO MOVE INTO THE
CLINIC, RIGHT?
IT COULD BE BOTH.
AND THEN SO DO YOU UNDERSTAND
WHO YOU ARE REACHING OUT TO AND
THEIR PRIORITIES AND THEIR
CAPABILITIES?
DO YOU UNDERSTAND THE DRUG
DEVELOPMENT PROCESS ITSELF?
AGAIN, THE BOT CAMP DO YOU HAVE
A REALLY GOOD UNDERSTANDING OF
WHAT IT TAKES TO GET TO GOAL AND
WHERE YOUR ORGANIZATION WANTS TO
FOCUS THEIR EFFORTS, RIGHT,
BECAUSE NONE OF US CAN DO
EVERYTHING.
SO IT'S REALLY, DO YOU HAVE, CAN
YOU FIND THAT MISSION MATCH,
RIGHT, AND THEN I THINK THE
CHALLENGE IS NAVIGATING THAT
ORGANIZATION THAT YOU ARE THEN
TRYING TO REACH OUT TO, RIGHT.
AND THAT CERTAINLY IS SOMETHING
THAT PFIZER HEARS A LOT OF,
RIGHT.
BECAUSE WE'RE SUCH A LARGE
COMPANY AND TRYING TO LOCK FOR
THOSE FOLKS WHO ARE FOCUSED ON
PATIENT ADVOCACY, WE'RE PROBABLY
BEING A GREAT PLACE TO START IN
TERMS OF THAT INITIAL CONTACT.
>> MAYBE I COULD SE EVEN ADD TO
THAT.
I WANTED TO GIVE THE PERSPECTIVE
FROM OUR FOUNDATION PERSPECTIVE,
WE DIDN'T -- WE WANTED AS MANY
PEOPLE AS POSSIBLE TO BE
INTERESTED IN OUR DISEASE AND SO
WE WERE REALLY QUITE AGNOSTIC TO
PRETTY MUCH ANY ASPECT OF THE
COMPANY.
I MEAN, WE WERE SUPER EXCITED
WITH COMPANIES THAT WERE LARGER
ESTABLISHED COMPANIES IN THE
SPACE AND TOOK A INTEREST IN FTD
BUT SMALL, GRAND NEW START UP
BIO TECHS THAT WERE INTERESTED
IN AND WE TRIED TO GIVE THEM
EQUAL ACCESS AND EQUAL
OPPORTUNITIES TO WHATEVER WE
COULD DO TO HELP THEM WAS OUR
MODEL BECAUSE WE DIDN'T KNOW WHO
MIGHT SUCCEED AND IN FACT, WE
HAD INTERESTING EXPERIENCES I
WOULD SAY OVER THE LAST 10 PLUS
YEARS WE'VE SPOKEN WITH 75
COMPANIES RANGING FROM ALL OVER
THE WORLD AND ALL KINDS OF
STAGES AND FUNDING LEVELS AND
SOMETIMES WE WOULD GIVE A
PRESENTATION AND THINK OH THAT'S
GOING TO GO NOWHERE AND THEN TWO
YEARS LATER THE COMPANY MIGHT
CIRCLE BACK AND SAY BY THE WAY,
HERE IS SOME DATA THAT WE HAVE
YOU JUST NEVER KNOW AND IN FACT
SOME OF THE COMPANIES, SOME
EARLY PLAYERS IN OUR SPACE
EVENTUALLY DROPPED OUT FOR A
VARIETY OF REASONS AND PLAYERS
WE DIDN'T KNOW HAD ACTIVE
PROGRAMS ARE FARTHER ALONG THAN
OTHERS SO I THINK THAT IN
HINDSIGHT IT'S OBVIOUSLY VERY
DEPENDENT ON THE RESOURCES AND
THE FOUNDATION BUT BECAUSE IT'S
SO UNPREDICTABLE WHO CAN GET A
DRUG INTO THE CLINIC, AND THEY
MIGHT COME YOUR WAY AS POSSIBLE.
>> AND IF ANYTHING COMES OUT OF
IT IMMEDIATELY YOU NEVER KNOW
WHAT HAPPENS IN THE FUTURE AND
IT'S GOOD FOR THEM TO KNOW ABOUT
YOU AND THE MORE RESOURCES THAT
YOU HAVE ABOUT YOUR SPECIFIC
DISORDER, THE BETTER EQUIPPED
YOU ARE TO HAVE THIS DIALOGUE
WITH A COMPANY AND TELL THEM WE
HAVE A REGISTRY AND A NATURAL
HISTORY STUDY . BANK, WHATEVER
YOU HAVE YOU KNOW THIS DISORDER
BETTER THAN ANYONE ELSE.
YOU ARE THE ONE THAT MIGHT
TRIGGER STARTING A PROGRAM IN
THIS COMPANY.
DO IT EARLY AND DO IT AS MANY
TIMES AS YOU CAN.
BIO MEETINGS, THE BIGGEST
PARTNERING MEETING FOR IN DOES
TREE.
IN THE PAST WE HARDLY SAW
PATIENT GROUPS AND THE LAST BIO
THERE WERE SO MANY PATIENT
GROUPS REQUESTING MEETINGS WITH
COMPANIES, IT'S AMAZING TO SEE
HOW MUCH ACTIVITIES THERE IS AND
HOPEFULLY THESE INTERACTIONS
WILL GENERATE AN ENERGY THAT
WILL START A PROGRAM AND HELP
SOMEONE START A PROGRAM SO DO IT
DEFINITELY.
>> THAT'S GREAT.
CAN YOU TALK ABOUT GROUPS AND WE
HAVE OUR SPECIFIC INTERESTS AND
WE THE ALIGNMENT HAS TO BE
BETWEEN A PATIENT GROUP AND A
GOAL WHICH IS CURE MY DISEASE AS
SOON AS POSSIBLE, RIGHT.
AND USUALLY YOU WORK WITH AN
ACADEMIC WHO NEEDS TO PUBLISH
AND GET GRANT MONEY AND TUN
YOU'RE ATENUREAT THE UNIVERSITYD
THEY NEED TO BE SUCCESSFUL AND
GENERATE REVENUE.
IT DOESN'T ALWAYS ALIGN.
MY RECOMMENDATIONS TO PATIENT
GROUPS THAT ARE WORKING WITH
ACADEMICS AND I KNOW THERE'S A
SESSION ABOUT WORKING WITH
ACADEMICS LATER SO I WON'T SPEND
TOO MUCH TIME AND GOING TO
PHARMA AND UNDERSTANDING THE
GOALS DON'T ALWAYS ALIGN BUT
THERE CAN BE ALIGNMENT IF
THERE'S VALUE BROUGHT IN BY THE
PATIENT GROUP OR BY THE
FOUNDATION THAT HAS ALL THE
INFORMATION THAT COULD HELP
ACCELERATE A PROGRAM OR WORKING
PARALLEL TO THE PRE CLINICAL OR
CLINICAL DEVELOPMENTS SO THIS
THINGS CAN BE EXPEDITED AND NOT
TROY TO DO IT STEP WISE ONE
AFTER THE OTHER BUT WORK
PARALLEL ON MANY THINGS.
ALSO, INTRODUCE THE COMPANIES TO
YOU AND YOUR FAMILY AND YOUR
STORY.
A LOT OF TIMES IT SWITCHES
SOMETHING IN THE BRAIN OF PERSON
THAT YOU ARE YOU CANNING TO AND
ALL OF A SUDDEN THEY UNDERSTAND
WHY THERE'S A SENSE OF URGENCY.
ON MY PH.D I WORKED ON DOWN
SYNDROME AND THE FIRST YEAR AND
THE LAB I'VE NEVER MET A FAMILY
THAT HAD A CHILD EFFECTED BY
DOWN SYNDROME AND NEVER MET A
PATIENT WITH DOWN SYNDROME AND
THE MOMENT IT HAPPENS, MY
PERSPECTIVE COMPLETELY CHANGED
AND A LOT OF TIMES, PEOPLE DON'T
KNOW AND HAVEN'T BEEN ABLE TO
GET THIS EXPOSURE SO HELP THEM
GET THAT EXPOSURE.
>> THAT'S REALLY IMPORTANT.
WHEN WE TALK ABOUT I.P. AND
LAURA, CAN YOU SHARE WITH US HOW
BLUE FIELD HAS DEALT WITH THE IP
ISSUES TO GET WORK ON THE FTD?
>> SO I THINK OUR FOUNDATION
FROM THE START, DECIDED THAT WE
WEREN'T GOING TO FUND PROGRAMS
WITHIN INDUSTRY AND SO THAT
MAKES IT A LOT EASIER IN THE
SENSE THAT YOU DON'T NEED TO
NEGOTIATE MORE COMPLICATED
CONTRACTS SO JUST THAT'S THE
STARTING POINT SO THEN WHAT WE
DID DO WAS WE FUNDED ACADEMIC
RESEARCH AND ESPECIALLY BECAUSE
WHEN OUR FOUNDATION WAS FOUNDED
THERE WAS LITTLE KNOWN ABOUT OUR
DISEASE OR THE MECHANISM OF WHY
MUTATIONS CAUSE FTD AND WHEN THE
ACADEMICS WERE SUCCESSFUL IN
CREATING TOOLS AS THEY DID THAT,
WHAT WE DECIDED TO DO WAS TROY
TO LOWER THE BARRIER TO GET
THOSE TOOLS INTO COMPANIES FOR
ANY COMPANY THAT MIGHT BE
INTERESTED IN WORKING ON FTD AND
SO, WE LICENSED SOME OF THE
TOOLS IN PARTICULAR MOUSE MODEL
AND PATIENT DERIVED CELL LINES
FROM DIFFERENT ACADEMIC
INSTITUTIONS WHERE INVESTIGATORS
HAD CREATED AND WORKED ON THEM
AND THEN WE HAD THE A LOT ABILIO
GET THEM IN THE HANDS OF
INTERESTED COMPANIES FOR EXPLORE
TORI RESEARCH AS QUICKLY AS
POSSIBLE BECAUSE OFTEN TIMES,
ACADEMIC INTERESTS, THEY MUST BE
BOGGED DOWN AND IN THIS WAY, WE
CAN HAVE A MASTER AGREEMENT THAT
WE CAN CONTROL THE PROCESS OF
SENDING OUT THE REAGENTS VERY
QUICKLY AND MAKE IT AS
STREAMLINE AS POSSIBLE AND ITS
DO THINK THAT HELPED.
IF COMPANIES HAD INITIAL
INTEREST AND THEY THOUGHT, MAYBE
I WOULD LIKE TO TROY AN IDEA OR
HYPOTHESIS, SO VERY, VERY EX MOR
EXPLORATORY RESEARCH AND IT
MIGHT JUMP START A PROGRAM SO WE
FELT THAT TIME WAS OF THE
ESSENCE AND THE WAY WE CAN DO
THAT WAS TO OFFER THIS SUB
LICENSING OPPORTUNITIES.
>> GREAT.
CATHERINE.
>> THAT'S BRILLIANT.
RIGHT, BECAUSE I CAN'T SPEAK IN
DETAIL AROUND THE COMPLEXITIES
OF IT BUT I DO KNOW THAT WHEN WE
TRY TO OPERATIONALIZE
COLLABORATIONS, THROUGH
CONTRACTING, WHETHER IT'S WITH
AN EPIDEMIC OR ADVOCACY GROUP,
THE ISSUES BECOME VERY, VERY
COMPLEX AND VERY FAST AND IT
DOES SLOW DOWN THE PR PROCESS TT
IDENTIFY AND ALIGN AGAINST A
SHARED SENSE OF URGENCY AND IT
BREAKS DOWN WHEN YOU TRY AND
EXECUTE THESE CONTRACTS BECAUSE
YOU HAVE VERY BUSY ACADEMIC
INSTITUTIONS AND YOU HAVE YOUR
LEGAL PARTNERS AT A COMPANY THAT
ARE REALLY WANTING THAT FREEDOM
TO OPERATE AND CLARITY.
>> MAYBE I CAN JUST CLARIFY, OUR
ABILITY TO SUB LICENSE WAS
REALLY FOR EXPLORATORY RESEARCH.
IF A COMPANY PERSUADE A PURSUE M
THEY NEED TO GET A COMMERCIAL
LICENSE.
WE REALLY JUST WANTED TO
INCENTIVIZE THAT FIRST STEP.
LET'S GET A HOOK INTO THE
COMPANY TO GET THEM INTERESTED
ON THAT PROGRAM AND THEN IF IT
GOES FORWARD, THEN THEY WILL
STILL HAVE TO DO THE COMMERCIAL
NEGOTIATIONS BUT LET'S GET THEM
A LITTLE BIT INTERESTED BEFORE
TO SEE WHETHER OR NOT IT'S WORTH
THEIR TIME.
>> I DO HEAR FROM SOME
FOUNDATIONS THAT THEY ARE NOT
GOING TO BOTHER WITH FILING I.P.
OR BOTHERING WITH I.P. BECAUSE
IT'S SUCH A DISTRACTION AND I
THINK IF YOU UNDERSTAND HOW
VALUABLE THIS INTELLECTUAL
PROPERTY IS IF YOU WANT TO
PARTNER WITH A COMPANY THAT WILL
LATER COMMERCIALIZE IT, IT'S
VERY IMPORTANT FOR THE PARTNER.
SO YOU MAY NOT THINK IT'S
IMPORTANT FOR YOU AND IT COST
MONEY TO FILE IP AND TO MAINTAIN
IT, IT IS IMPORTANT IF YOU
PARTNER WITH A COMPANY THAT
WANTS IT GENERATE REVENUE AND
HAVE AN ADVANTAGE WITH IT GETS
TO THE MARKET WITH A PRODUCT
THAT'S VERY IMPORTANT TO HAVE
I.P. SO DON'T UNDERESTIMATE THE
VALUE OF IT AND FOR PATIENT
GROUPS THAT ARE WORKING WITH
ACADEMICS AND FUNDING RESEARCH,
IT'S VERY IMPORTANT THAT YOU GET
ACCESS TO THAT I.P. THAT IS
FILED BASED ON THE MONEY THAT
YOU FUNDED THE PROGRAM WITH AND
LICENSE THIS I.P. WHEN THE
RESEARCH IS DONE AND THE RESULTS
LOOK PROMISING BECAUSE IF YOU
FUNDED IT, YOU DESERVE TO
CONTROL IT EVER AT RESEARCH IS
DONE.
>> YEAH, I'M ASSUMING THIS I.P.
SECTION WILL BE DISCUSSED THERE
AS PART OF THE BOOTCAMP.
>> OH, YEAH.
WE STUDY HALF A DAY ON THE
BUSINESS OF SCIENCE WHICH IS
INTELLECTUAL PROPERTY AND
NEGOTIATING LICENSES WITH THE
UNIVERSITIES AND WHAT CAN YOU
ASK FOR AND HOW TO STRUCTURE AND
WE'VE SEEN SEVERAL GROUPS THAT
FUNDED RESEARCH WITHOUT GETTING
ACCESS TO THE RESULTS OF THE
RESEARCH AND THE FRUIT THAT CAME
OUT THE RESEARCH AND IT WAS
FRUSTRATING TO THEM.
>> OH MY GOSH.
THAT'S A GOOD SEG WAY TO MY NEXT
QUESTION WHICH IS YOU GUYS HAVE
BEEN IN THIS SPACE AND THE
SCIENCE WIELD FIELD FOR A LONG
TIME.
YOUR WHOLE CAREERS.
I WANT INFORMED ASK YOU, YOU
KNOW, IF YOU COULD THINK OF SOME
CASES WHERE THE RELATIONSHIPS
HAVE WORKED REALLY WELL BETWEEN
PATIENTS GROUP AND INDUSTRY.
NO NAMES BUT WHAT ARE SOME CASES
THAT YOU MIGHT CITE AS SORT OF
MODELS FOR GOOD RELATIONSHIPS IN
PARTNERSHIPS?
>> I HAVE A GREAT EXAMPLE
BECAUSE A PRESS RELEASE JUST
CAME OUT YESTERDAY AND THAT IS A
RELATIONSHIP BETWEEN
ULTRA-GENICS AND THE ANGLE
FOUNDATION AND THE COMPANY THE
FOUNDATION SPUN OUT AND IT'S A
RELATIONSHIP THAT STARTED OVER W
YEARS AGO ULTRA-GENICS SUPPORTED
THE GENETICS, THE COMPANY SPUN
OUT OF THE FOUNDATION AND
ULTRA-GENICS ENTERED A
COLLABORATION FOR GENETICS AND
FUNDED SOME OF THE RESEARCH THAT
THEY DID AND TOOK IT INTO THE
CLINIC WITH AN OPTION TO ACQUIRE
THE COMPANY AND THEY ENDED UP
YESTERDAY ANNOUNCING THAT THE
COMPANY WAS ACQUIRED SO IT TOOK
TIME AND IT WAS A VERY
INTERESTING COLLABORATION FROM
WHEN WE SIGNED THE AGREEMENT AND
THE ACQUISITION OF THE COMPANY.
IT WAS A VERY PROLIFIC
RELATIONSHIP AND ENDED WITH
HOPEFULLY WITH A THERAPY IN THE
CLINIC AND WE'LL GET TO MANY
PATIENTS SO I THINK THE LEARNING
FROM IT AND NOT THAT I RECOMMEND
EVERY FOUNDATION TO SPIN OUT
COMPANIES AND NOT EVERY
FOUNDATION CAN DO IT, YOU NEED
RESOURCES FOR IT BUT IT GOES
BACK TO WHAT I SAID BEFORE,
ENGAGE EARLY WITH A PHARMA
PARTNER AND GET AS MUCH
INFORMATION AS YOU CAN AND TRY
TO GET THEM TO HELP WITH
EXPERTISE, EVEN IF THEY'RE NOT
INVESTING MONEY, SOMEONE YOU CAN
TAP INTO AND ASK QUESTIONS AND
MAKE THEM AWARE OF YOUR PROGRAM
AND GET THEM TO LIKE THE PROGRAM
AND EVENTUALLY IT MIGHT END WITH
A GREAT BUSINESS RELATIONSHIP AS
WELL.
>> AND THAT DISEASE IS
ULTRARARE, RIGHT.
LIKE WHAT IS?
>> THERE ARE A FEW THOUSAND
PATIENTS IN THE WORLD AT THE
MOMENT.
>> HOW LONG DID IT TAKE TO GET
FROM STARTING A BUSINESS TO THE
ACQUISITION?
>> SO THEY STARTED THE COMPANY
IN 2019 OR 2018.
AFTER THEY SHOPPED IT AROUND
WITH MANY IN THE STREET AND
PARTNERS AND THERE WAS AN
INTEREST AT THE TIME BECAUSE
THEY HAD VERY LOW DATA SO THEY
DECIDED TO CONTINUE FUNDING THE
PROGRAM AND THEY GOT A FULL AND
VERY COM ROW HENCE I HAVE PROOFD
SO THEY DECIDED TO LOOK FIRE
PARTNER AS WELL AND THEY
ACTUALLY WERE LOOKING FOR
VENTURE FUNDING OR A PARTNER AND
WE MET WITH THEM AND WE LIKED
THE PROGRAM AND WHAT WAS
PRESENTED TO US AND DECIDED TO
ENGAGE IN THIS COL A COLLABORAT.
THEY START INSIDE 2017 AND WE
STARTED THE RELATIONSHIP WITH
THEM IN 2019 AND THREE YEARS
LATER THE COMPANY WAS ACQUIRED.
>> AMAZING.
AMAZING.
ANY OTHER SUCCESS STORIES YOU
CAN THINK OF?
>> I'M HAPPY SHARE AN EXPERIENCE
BECAUSE I THINK IT MIGHT BE A
DIFFERENT APPROACH TO
CONSIDERING WHAT SUCCESS LOOKS
LIKE, RIGHT.
TO THE COMMENTS OF MY PANEL
COLLEAGUES EARLIER, I THINK OUR
GOAL WHERE I SIT IN MY
ORGANIZATION IS REALLY FOCUSES
ON THAT DISCOVERY PIECE AND
CONCEPTS SO OUR MODEL IS TO
ENGAGE VERY EARLY ON TO CO
CREATE THE RESEARCH QUESTIONS,
RIGHT, BACK WHERE ADVANCED
HOPEFUL LOW THERAPEUTIC
DEVELOPMENT AND I THINK THE CON
SEX OF WORKING WITH A PARTICULAR
PATIENT SAD ROW KA SEE GROUP WAS
REALLY FOCUSED ON A SPECIFIC
RESEARCH QUESTION WITH A DEFINED
OUTCOME THAT COULD TIE TO A
DECISION, RIGHT.
OBVIOUSLY YOU DON'T KNOW WHAT
THE OUTCOME IS GOING TO BE AT
THE BEGINNING BUT YOU KNOW
REGARDLESS OF OUTCOME YOU ARE IN
A POSITION TO MAKE A DECISION
AND THE PROJECT THAT WAS CO
FUNDED, YOU KNOW WAS A TARGET
IDENTIFICATION SCREENING AND FOR
THE ADVOCACY ASSOCIATION, THE
OUTCOME WOULD GIVE THEM
CONFIDENCE IN HOW THEY WERE
GRANT FUNDING GIVEN A PARTICULAR
MECHANISTIC APPROACH TO
ADDRESSING THEIR CONDITION.
FOR PFIZER, IT WAS YOU KNOW, DO
WE PURSUE THIS MECHANISTIC
APPROACH OR NOT?
AND SO THERE WAS CLARITY ON WHAT
WAS THE RESEARCH QUESTION AND
WHAT DECISIONS COULD ENTITIES
MAKE GIVEN THE OUTCOME AND I
THINK WE WEREN'T ABLE TO
IDENTIFY ANY HITS AND I THINK
THE CONCERN THERE WAS HOW IS
THIS GOING TO IMPACT THE
RELATIONSHIP BUT WE ALREADY
DISCUSSED THE POTENTIAL OUTCOMES
SO FOR THE ASSOCIATION, THEY
WERE VERY SATISFIED BECAUSE NOW
THEY KIND OF COULD STRATEGICALLY
REORIENT THEIR STRATEGY AROUND
GRANT FUNDING, RIGHT, AND PHASE
PFIZER HADCLARITY AND COULD REPE
SOME PROGRAMS FOR THAT
INDICATION AS WELL AND SO WAS IT
SUCCESSFUL IN FINDING A HIT, NO,
BUT IT WAS SUCCESSFUL IN
ANSWERING THE RESEARCH QUESTIONS
AND SUSTAINING THAT RELATIONSHIP
BECAUSE AGAIN YOU WERE IN THERE
ALIGNED WITH THE SHARED PURPOSE.
>> GREAT, LAURA, ANY STORIES YOU
CAN SHARE?
>> WELL, I GUESS JUST GENERALLY
I WOULD SAY THAT I THINK THERE
ARE THESE PRACTICAL -- THE
CONCEPT OF OUR NEED TO HAVE A
STRONG RELATIONSHIP WITH THE
COMPANIES IS SO PARAMOUNT AND
SOMETIMES IT CAN BE FRUSTRATING
WHEN IT DOESN'T WORKOUT AND I
THINK THAT THERE ARE SO MANY
DIFFERENT FACTORS AND INFLUENCES
FROM THE PRACTICAL SUCH AS HOW
MANY PATIENTS HAVE THIS DISORDER
AND HOW MUCH MONEY MIGHT HAVE
FOUNDATION CONTRIBUTE AND TO
REALLY THESE INTANGIBLES LIKE
INTERPERSONAL RELATIONSHIPS
WHICH ARE HARD TO DEFINE.
THEY CAN BE VERY IMPORTANT AND
HAVING A CHAMPION WITHIN A
COMPANY FOR YOUR DISORDER CAN BE
REALLY, REALLY TRANSFORMATIVE
BECAUSE AS A COMPANY LOOKED AT
REPORT PORTFOLIO OF ALL THE
DIFFERENT THINGS THEY'RE WORKING
ON, HAVING A VOCAL AND
COMPELLING CHAMPION WHEN YOU
HAVE FIVE PROJECTS THAT ARE KIND
OF HAVING TROUBLE AND THE
COMPANY IS TRYING TO DECIDE
WHERE TO DOU DEVOTE RESOURCES SO
DEVELOPING THOSE PERSONAL
RELATIONSHIPS WHICH IS EASIER
SAID THAN DONE BUT I DO THINK
THAT IS AN IMPORTANT COMPONENT
TO CONSIDER.
>> GREAT.
THANK YOU.
I'M GOING TO ENCOURAGE OUR
AUDIENCE NOW TO START PUTTING
THEIR QUESTIONS IN THE CHAT.
AS WE WAIT FOR PEOPLE TO PUT
THEIR QUESTIONS IN, I WANT TO
ASK THE OTHER SIDE OF THE
QUESTION I ASKED, WHICH IS,
AGAIN, NO NEED FOR -- TO GO ABLE
TO TAKE THE PROGRAM AND EITHER
CONTINUE DEVELOPING AND DO TON
YOUR OWN OR PLACE IT AND THE
FATE OF THE PROGRAM IS NOT
POSSIBLE AND NOT EVERY COMPANY
IS HAPPY TO DO THIS BUT IT HELPS
IF YOU ARE ABLE TO NEGOTIATE
THIS SO THERE HAVE BEEN CASES
WHERE THIS WAS AND DON'T HAVE
ANY CONTROL OVER THE PROGRAM IF
IT'S SHELVED AND PEOPLE DON'T
WANT TO HAND IT OVER TO SOMEONE
ELSE FOR COMPETITIVE REASONS AND
IS IT JUST DIES A SLOW DEATH AND
THAT'S REALLY THE WORST-CASE
SCENARIO SO THE SECOND THING IF
YOU ARE PARTNERING WITH A
COMPANY TO TRY TO UNDERSTAND
WHERE YOUR PROGRAM IS AND IN
THEIR PIPELINE IS IT GOING TO BE
THE FIRST OR SECOND OR THIRD
PRIORITY OR IS IT GOING TO BE
THE FIFTH OR 2:00 PLACE IN THE
LIST AND NOW WHEN THE MARKET WAS
GREAT A LOT OF PEOPLE HAD REALLY
PIPELINES AND NOW WHEN TIMES ARE
HARDER AND PEOPLE NEED TO GET
BACK IF YOU ARE NUMBER 15 THEY
ARE HIGHER WHEN THE TIMES ARE
GREAT AND THERE'S A LOT OF
RESOURCES AROUND.
>> AGREE OF EVERYTHING.
WHEN YOU THINK ABOUT
RELATIONSHIPS FAILING, WHETHER
IT'S BETWEEN AN ADVOCACY GROUP
AND AN INDUSTRY OR HUSBAND AND
WIFE, RIGHT, IT BREAKS DOWN
AROUND COMMUNICATION AND
MANAGING EXPECTATIONS, RIGHT.
IF YOU ARE SOMETIMES OUR
CHALLENGE IS NOT BEING DIRECTED
AND NOT SAY HERE IS THE PRIORITY
OF THIS PROGRAM AND THIS IS THE
STAGE IT'S AT AND THIS IS WHAT
WE NEED TO DEMONSTRATE TO MOVE
IT FORWARD AND THIS IS WHERE IT
SITS IN RELATIONSHIP TO
RESOURCES ACROSS THE COMPANY AND
WE MIGHT TEND TO SAY WE'RE
COMMITTED TO THIS DISEASE AND
WE'RE IN IT FOR THE LONG HALL
AND WHEN AND THE OTHER PARTNER.
>> LAURA DO YOU HAVE ANYTHING
ELSE TO ADD TO THAT?
>> WE'RE STILL WAITING ON
QUESTIONS BUT THERE'S A QUESTION
FROM DARLENE.
SHE SAID AND I DON'T KNOW IF
THERE'S SHOULD THEY FUND A
SEPARATE MOUSE.
>> YEAH, I THINK IT'S A GREAT
QUESTION.
FIRST TRY AND UNDERSTAND WHY HE
IS NOT WILLING TO SEND OUT THE
MOUSE AND MAYBE IT'S SOMETHING
RELATED TO HIS PUBLICATION AND
HE WANTS TO BE ON THE
PUBLICATIONS OR HE WANTS FUNDING
AND THERE'S ALWAYS THE REASON
WHY THIS IS NOT WHAT PEOPLE ARE
NOT WILLING TO SHARE AND TRY TO
UNDERSTAND BETTER AND MAYBE
ADDRESS THE SPECIFIC REASONS IF
NOT IF THERE'S NO OTHER CHOICE
AND NO OTHER MOUSE MODEL THEN
YEAH THEY WOULD.
YOU HAVE TO MAKE YOUR OWN MOUSE
MODEL THAT TAKES TIME AND IT
COST MONEY AND IT'S DEFINITELY
WORTH HAVING CONTROL OVER A KEY
COMPONENT OF ANY RESEARCH PLAN
THAT YOU ARE GOING TO BE
FUNDING.
>> YEAH.
THAT IS REALLY HARD.
LAURA, YOU GUYS HAVE DONE A
GREAT JOB.
I KNOW YOU MENTIONED YOU HAVE A
SUNSET DATE OF 2025, YOU GUYS
HAVE DONE A REALLY GREAT JOB OF
LEVERAGING FUNDING FROM OTHER
SOURCES AND YOUR PARTNERSHIPS,
CAN YOU TALK ABOUT THAT SUNSET
PROVISION AND ARE YOU GUYS
STICKING TO THAT?
I MEAN YOU HAVE FOUR DRUGS NOW
GOING INTO CLINICAL TRIAL BUT
CAN YOU TALK A LITTLE BIT ABOUT
HOW YOU LEVERAGED YOUR
PARTNERSHIPS, ESPECIALLY IN THE
FUNDING SIDE?
>> SURE, I DO THINK IT UNUSUAL T
OUR FOUNDERS WERE INTENTIONAL IN
IT BECAUSE IT FORCES US TO BE
HIGHLY FOCUSED AND I THINK TO
MAKE SOME -- I THINK LAURA
FROZE.
HOPEFULLY SHE CAN COME BACK.
>> YES, EVERYONE ELSE SEEMS
FINE.
>> OK.
>> AS WE WAIT FOR LAURA TO COME
BACK, LET'S JUST MOVE ON TO THIS
QUESTION ON WHAT CAREGIVERS AND
PATIENTS CAN DO TO ADVANCE
SCIENCE OUTSIDE OF FUNDING
FOUNDATIONS.
TIME IS MONEY TO INDUSTRY AND I
THINK THAT WE HAVE INDIVIDUALS
AND SHE'S REPRESENTING ALS AND
THEY HAVE A GREAT DEAL TO OFFER
TO INDUSTRY THAT CAN HELP
THERAPY DEVELOPMENT MOVE FORWARD
FASTER.
I THINK CATHERINE, YOU MIGHT BE
ABLE TO ANSWER THAT QUESTION.
>> YOU KNOW, IT DOESN'T HAVE TO
BE A FUNDING AND IT'S TO
IDENTIFY WHAT ARE SOME OF THE
BARRIERS THAT ARE IN PLACE TO
DRUG DEVELOPMENT AND MAYBE IT'S
AN ISSUE OF ACCESS TO DATA RIGHT
AND I THINK THAT THE QUESTION
WAS ACCESS TO A MOUSE MODEL,
WELL, YOU KNOW, I THINK WE ALL
EXPERIENCE CHALLENGES WHERE SOME
DATA IS SILOED AND IT'S REALLY
HARD TO ACCESS IT AS AN INDUSTRY
SPONSOR AND WHEN WE HAVE TO
UNDERSTAND WHAT IS NATURAL
HISTORY AND SO ARE THERE THINGS
THAT YOU AS AN ORGANIZATION CAN
BE DOING THAT ENABLED THAT IN
YOU ARE JOINING CO CONSORTIA SOT
DOESN'T HAVE TO BE SPECIFICALLY
FUNDING BUT A GOOD SENSE OF SOME
OF THE EXISTING CHALLENGES IN
THE RESEARCH DEVELOPMENT SPACE
FOR YOUR COMMUNITY.
>> GREAT.
LAURA WE LOST YOU FOR A LITTLE
BIT.
I DON'T KNOW WHERE ENDED.
>> I'M SORRY.
WORKING WITH OTHER FOUNDATIONS
IF YOU HAVE THEM IN YOUR SPACE
IS GREAT.
THAT WAS MY POINT.
LET'S TAKE THIS QUESTION FROM
SARAH FROM THE SPINA BIFIDA
ASSOCIATION.
AND SHE SAID OUR ORGANIZATION
PUBLISHED OUR FIRST COMMUNITY
AND THEY WANT ANSWERED AND IT'S
CONSIDERED AND COME SHOULD
PATIENT GROUPS DO FIRST?
DO YOU WANTED TO RESTATE THAT
FOR THIS?
>> I'M SORRY, I THOUGHT LAURA
WAS ADDRESSING THAT QUESTION.
>> ANYONE.
LAURA, YOU CAN.
I THINK IS MAYBE A LITTLE UNDER
APPRECIATED AND UNDERSTANDING
THE SINCE IS OF COURSE THE
FOUNDATION OF ANY DRUG AND IT'S
EASIER TO ADDRESS AND ALTHOUGH
WE CERTAINLY FEEL LIKE WE HAVE
DONE A LOT TO INFLUENCE DRUG
DEVELOPMENT IN THE SPACE WE
REALIZE BECAUSE OF THE MECHANISM
OF DISEASE MUTATIONS CAUSE A
DEFICIENCY SO IT'S STRAIGHT FORD
THERAPEUTIC APPROACH INDIVIDUALS
HAD HALF THE AMOUNT OF NORMAL
THAN THEY SHOULD SO IT'S A
REPLACEMENT STRATEGY AND
INCREASING REALLY VERY STRAIGHT
FORWARD AND HOPEFULLY, RIGHT AND
I THINK THAT IT HAS BEEN
ENORMOUS BENEFIT AND SO I THINK
SOMETIMES UNFORTUNATELY, IT'S
JUST A LONGER STRUGGLE IF IT'S
NOT A CLEAR STRAIGHT FORWARD
THERAPEUTIC APPROACH OR IF
THERE'S NOT AS MUCH BASIC
SCIENCE UNDERSTOOD ABOUT THE
MECHANISM OF DISEASE AND THESE
THINGS TAKE TIME.
FUNDING BASIC SCIENCE AND
DISCOVERY RESEARCH AS NECESSARY
TO HELP MAKE CLEAR PER
THERAPEUTIC APPROACHES.
>> WE HAVE ANOTHER QUESTION
HERE.
WE HAVE A QUESTION AT END POINTS
AND DO YOU HAVE ANY SUGGESTIONS
ABOUT HOW TO PER WAYED INDUSTRY
TO ACCEPT CLINICAL END POINTS
WITH PATIENTS AND CAREGIVERS,
OTHER THAN WHAT THEY, THE
INDUSTRY MIGHT BELIEVE ARE THE
APPROPRIATE END POINTS.
>> YES, THERE'S A BALANCE AND A
DISCUSSION BETWEEN THE END
POINTS THAT ARE MEANINGFUL FOR
PATIENTS, RIGHT, AS DEFINED BY
WHAT YOU HAVE TO DEMONSTRATE BY
THE FDA AND HOW THEY CAN BE
MEASURED AND THAT IS SOMETIMES A
CHALLENGE.
AND THEN THE LANGUAGE OF DATA
ABOUT THE END POINT THAT THE FDA
IS GOING TO ACCEPT.
>> ASSUME CERTAIN RISKS IN ORDER
TO GET A BENEFIT FROM AN
INVESTIGATIONAL THERAPY AND THE
TRADE OFFS IS THEY WILL USE
PERHAPS, IN SOME OF THOSE
CLINICAL END POINTS SO DOING
SOME OF THAT STRUCTURED RESEARCH
TO SAY, THIS IS WHAT THE
COMMUNITY IS WILLING TO DO.
RESPONSORS WILL LOOK AT THAT AND
SAY OH MY GOSH, I HAVE NO IDEA
AND IN TERMS OF PREFERENCES WHAT
I'M PRO FOESING IS SO FAR AT THE
BOTTOM OF THE LIST AND AND
REGULATORS WILL SAY THE
COMMUNITY IS SPEAKING AS A WHOLE
AND WE CAN SEE A DIFFERENT RISK
BENEFIT UNDERSTATED PATIENT
PREFERENCE RESEARCH, RIGHT, SO
KIND OF COUPLE POINTS TO THAT
QUESTION.
>> YOU COVERED EVERYTHING I
WANTED TO SAY BUT THE
PATIENT-FOCUSED MEETINGS ARE
USEFUL FOR THAT SO IF YOU HAVE
DONE YOUR RESEARCH AND YOU HAVE
INVESTIGATED THE COMMUNITY ON
WHAT END POINTS WOULD MATTER TO
THEM AND WHAT THE RISK IS
THEY'RE WILLING TO TAKE, WHEN
YOU TALK TO THE FDA YOU BRING IT
UP IN THESE PATIENT FOCUSED
MEETING AND FAMILIARIZE THEM
WITH YOUR LIMITS AS A PATIENT
GROUP AND IT'S DIFFICULT BECAUSE
WITHIN A PATIENT GROUP, THERE
WILL BE FAMILIES THAT WILL TAKE
MORE RISK THAN OTHERS SO THEY
WILL ENROLL FIRST IN THE CLIP
CAL TRIALS?
PROBABLY.
BUT WHAT HAPPENS WITH ALL THE
OTHER FAMILIES THAT ARE NOT
WILLING TO TAKE THAT RISK?
IT'S A BIG DISCUSSION YOU ARE
TALKING ABOUT WHICH IS ONE AND
DONE AND YOU CAN'T REVERSE IT
AND THE BENEFITS YOU GET WITH IT
THERE MIGHT BE SOME OTHER THINGS
THAT YOU GET WITH IT AS WELL BUT
MAY NOT HAVE SIGNED UP FOR.
YOU DID SIGN UP FOR BUT HOPING
IT WON'T HAPPEN.
>> WE HAVE JUST A COUPLE MINUTES
LEFT.
AND I HAVE TWO QUESTIONS FOR
YOU, ONE, AND EACH OF YOU CAN
ANSWER THIS, THE GREATEST
MISCONCEPTION THAT YOU THINK A
PATIENT GROUPS HAVE OF INDUSTRY
AND INDUCE TREMENDOUS HAS I
PATIENTS AND YOUR LAST TAKEAWAY
YOU WOULD LIKE TO SHARE AND
ADVICE YOU WOULD GIVE PATIENT
ADVOCACY GROUPS SEEKING WITH
STRONG RELATIONSHIP WITH PHARMA
AND INDUSTRY.
>> ANYONE CAN START.
LAURA, WHY DON'T YOU START.
>> I GUESS THE MISCONCEPTION
WOULD BE PERHAPS I DO THINK
THERE CAN BE A PERCEPTION OF
PHARMA BEING SOLELY MOTIVATED BY
MONEY AND ALTHOUGH OF COURSE
THEY'RE A MONEY-MAKING
ENTERPRISE THEY'RE VERY HUMAN
SCIENTISTS WORKING WITHIN PHARMA
WHO ARE DEDICATING THEIR LIVES
AND WORKING VERY LONG HOURS TO
HELP CREATE DRUGS TO HELP FIGHT
DISEASES AND I THINK MUCH TIMES
ONE TO ONE BUILT THESE
RELATIONSHIPS WITH FARTHER SUED
CAL SCIENTIST TO SEE THEIR LEVEL
OF DEVOTION AND THE WORK THAT
THEY DO IS REALLY GRATIFYING AND
I THINK FROM THE SECOND QUESTION
ABOUT WHAT IS THE BIGGEST
ADVICE, I THINK NOT TO UNDER
VALUE YOUR OWN CONTRIBUTIONS AND
WHAT YOU CAN DO AND TO GET
STARTED AS EARLY AS POSSIBLE ON
CREATING PATIENT REGISTRIES AND.
>> IT COMES FROM THE PATIENT
GROUPS THAT THIS IS DEFINITELY
CHANGING AND PEOPLE LIKE MYSELF,
WE'RE A SMALL COMPANY AND WE ALL
KNOW THE VALUE OF WHAT THE
PATIENT GROUP HAS BEEN THROUGH
THE TABLE BUT A BIG COMPANY LIKE
PFIZER HAVING A PERSON LIKE
CATHERINE, AND OBVIOUSLY SHE
UNDERSTANDS THE VALUE AND SHE IS
THE SEG WAY INTO THE COMPANY IN
MANY CASES AND IT'S THE GREAT
JOB THAT EACH AND EVERY ONE OF
YOU ARE DOING IN MAKING SURE WE
UNDERSTAND WHAT EXPERTISE YOU
BRING TO THE TABLE.
>> LAST ADVICE, KATHRYN.
>> OH MY GOODNESS, I WOULD SAY,
I'M STILL REFLECTING ON THE
MISCONCEPTIONS AND AGREE THAT
INDUSTRY TENDS TO UNDER
APPRECIATE THE EXPERTISE IN THE
PATIENT COMMUNITY AND IN
REVERSE, THERE'S PROBABLY A
MISCONCEPTION AROUND INDUSTRY
THAT WE'RE KIND OF WITHHOLDING
INFORMATION AND WE KNOW THAT THE
PATH WAY WITH CERTAINTY IN
DEVELOPING A DRUG AND THAT'S
JUST NOT THE CASE BECAUSE YOU
KNOW, THE INTEREST IS REALLY
EMPLOYING INNOVATIVE MECHANISMS
AND DRUGS WHERE THE PATH ISN'T
MAPPED OUT FOR US AND WE'RE ALL
KIND OF LEARNING AS WE GO AND
APPLYING OUR EXPERTISE BUT IT'S
NOT SOME MAGIC BULLET THAT WE'RE
HOLDING ON TO AND NOT PAYING
ATTENTION TO.
THANK YOU FOR THIS WONDERFUL
INSIGHTFUL DISCUSSION.
THANK YOU FOR ATTENDING OUR
SESSION AND WE LOOK FORWARD TO
THE NEXT.
THANK YOU, EVERYONE.
>> THANK YOU ALL SO MUCH FOR
MODERATING THAT FANTASTIC
SESSION.
SO, WITH THAT, I AM GOING TO
TAKE US INTO OUR NEXT SESSION
WHICH IS HOW TO FIND INNOVATIVE
METHODS TO FUND RESEARCH IN AN
EFFORT TO SUPPORT SCIENCE AS A
SMALL BUSINESS SO THIS SESSION
WILL BE MODERATED BY OUR
NON-PROFIT FORM EXECUTIVE ME
GERALDINE BLISS.
THE PRESIDENT AND CO-FOUNDER OF
CURESHANK, A NON-PROFIT
ORGANIZATION WITH ACCELERATED
DRUG IN PMS AND RELATED SHARINGK
DISORDERS.
SHE SERVED ON THE BOARD OF
DISTRICTORS FOR TWO YEARS AND
RESEARCH CHAIRPERSON FOR SEVEN.
AND THAT ROLE SHE CHAIRED THE
FIRST SCIENTIFIC STRATEGIC
PLANNING INITIATIVE SUCCESSFULLY
A REPLIED FOR GRANTS TO FURTHER
PMS RESEARCH SUPPORT INITIATIVES
AND ORGANIZED SIM POSIA IN 2012,
14 AND 18 A MEETING IN '13 AND
MULTIPLE WORKSHOPS ON PMS AND
THE 2016 ASK 2018 AND FAMILY
CONFERENCE RESEARCH SESSION THIS
IS 2010, 2012 AND 2014 AND
ESTABLISHED A PMS RESEARCH
REPOSITORY PARTNERSHIP WITH
AUTISM BRAIN AND THE NINH AMONG
MANY OTHER ACCOMPLISHMENTS I'M
NOT SURE WHEN SHE SLEEPS AND SHE
HAS SERVED AS A LAY REVIEWER FOR
THE FDA AND HAS A BACHELORS FROM
TRINITY AND MASTER FROM THE
UNIVERSITY OF HOUSE ON AND HER
PERSONAL MISSION IS TO HAVE
CURES FOR PMS, SHE AND HER
HUSBAND BRAD HAVE TWO SONS,
CHARLES AND NATHAN AND CHARLES
DOES HAVE PMS CAUSED BY THE
SHANK 3 GENE SO WITH THAT,
PLEASE TAKE IT AWAY FOR OUR NEXT
SESSION.
>> GREAT, THANK YOU FOR THAT
NICE INTRODUCTION.
FOR THOSE OF YOU WHO
PARTICIPATED IN THE NINDS
NON-PROFIT FORUM LAST YEAR, YOU
MIGHT REMEMBER ALISON WHO GAVE A
TALK ABOUT THE FOUNDATION FOR
PMS THERAPEUTICS AND THE COMPANY
THEY STARTED CALLED GENETICS AND
THEY ULTIMATELY PARTNERED WITH
YOU WILL INTEREST GENICS TO
DEVELOP A ASL TREATMENT FOR
PATIENTS AS YOU JUST HEARD
MENTIONED IN THE LAST PANEL,
YESTERDAY, ULTRA-AGA IT WAS ACQP
FRONT PLUS ROYALTY PAYMENTS SO
IT'S A REALLY EXCITING OUT AND
AND JUST REALLY SUPER EXCITING
FOR THE COMMUNITY.
SO TODAY, WE'RE GOING TO
CONTINUE THIS DISCUSSION ABOUT
HOW NON-PROFIT GROUPS CAN
SUPPORT SCIENCE AS A SMALL
BUSINESS AND HOW TO FIND
INNOVATIVE METHODS TO FUND
RESEARCH AND OUR FIRST SPEAKER
IS JILL WOOD FROM PHOENIX NEST.
JILL IS THE FOUNDER AND C.E.O.
PHOENIX NEST WHICH IS THE
BIOTECH COMPANY FOCUSED ON
TREATING THE SYNDROME AND YOU
MIGHT KNOW JILL AS THE MOM TO
JONAH AND THE FOUNDER OF JONAH
JUST BEGUN A NON-PROFIT
ORGANIZATION TO RAISE AWARENESS
IS SUPPORT FOR INDIVIDUALS.
JILL WILL TALK TO US ABOUT
PHOENIX NEST AND HER SUCCESS AND
OBTAINING SMALL BUSINESS FUNDING
FROM THE NINDS.
CAN WE GO AHEAD AND GET JILL
STARTED, THEN.
>> I UNMUTED MYSELF.
I'LL TAKE IF FROM HERE.
THANK YOU FOR THAT INTRODUCTION.
AND THANK YOU FOR HAVING ME HERE
TODAY.
I AM JILL WOOD AND I AM THE
C.E.O. OF PHOENIX NEST AND I AM
JONAH'S MOM AND MY SON JONAH HAS
TYPE C YOU MIGHT HEAR ME REFER
IT AS SAM C AND I'LL REFEX TO P
PHOENIX NEST AS PN.
I'LL GIVE YOU A BACKGROUND ON
HOW PN CAME TO BE AND WORKING
WITH THE NINDS TO SUPPORT
PHOENIX NEST PROGRAMS.
SO, MY OBLIGATORY STACKS AND
THERE ARE FOUR DIFFERENT SUB
TYPES CAUSED BY MUTATION FOR
DIFFERENT GENES AND THERE ARE
THE INCIDENTS RATES ARE
ARBITRARY NO ONE REALLY KNOWS
AND THE TRUE INCIDENTS OF
SANPHILLIPPEO, THEY'RE RARE AND
TYPE C AND D BEING THE RAREST
TOGETHER THEY'RE OCCUR 1/70,000
PATIENTS.
BUT WHAT WE DO KNOW ABOUT
SANFILIPPO IT'S OF A STORAGE
DISEASE BECAUSED BY A SINGLE
GENE DETECT AND NO APPROVED
TREATMENTS FOR THE SUB TYPES.
SAN PHILLIPPE OWE WE STARTED WIS
JUST BEGUN AND WE DID CLOSE OUT
AND SENT OUR FAMILIES AND
SUPPORTERS THE SANFILIPPO
FOUNDATION SO I CAN FOCUS ON
PHOENIX NEST.
WHEN JOE NA WAS DIAGNOSED WE
FOUND THERE WAS NO TREATMENTS
AND THERE WAS NO ONE EVEN
WORKING ON A GRAHAM FOR SAN SO
WE CREATED JJB AND FOUND OUR
RESEARCHES AND CREATED A MOUSE
MODEL AND JOINED FORCES WITH THE
FAMILIES AROUND THE WORLD.
TOGETHER WE CREATED A TYPE C
SPECIFIC CONSORTIUM HAND
H.A.N.D.S. AND PERFORMED PRE
CLINICAL RESEARCH AND FOUNDED
THE FIRST AND ONLY GLOBAL
PATIENT REGISTRY CONNECT MPS.
I DON'T HAVE TO TELL THE
FAMILIES JUST HOW HARD IT IS TO
START FROM SCRATCH BUT FOR THOSE
OF YOU THAT DON'T KNOW, DOING
THIS AND GOING THROUGH TH THIS
JOURNEY WILL BE THE HARDEST JOB
OF YOUR LIFE.
NOTHING IS HARDER THAN WATCHING
YOUR CHILD DIE.
I'VE WILL BE DOING THIS FOR 12
YEARS NOW AND IF THINGS GO
PERFECTLY, I MIGHT BE IN THE
CLINIC IN ANOTHER FOUR YEARS.
MY SON WILL BE 18 BY THEN AND I
KNOW THAT HE MIGHT NOT MAKE IT.
I DO KNOW THAT JONAH'S LEGACY
WILL LIVE ON IN THE LIVES OF
CHILDREN THAT HE HAS SAVED.
PHOENIX NEST IS A TWO-MAN SHOW
THAT'S MY PARTNER IN CRIME THERE
WHO I HAVE TO GIVE CREDIT FOR A
LOT OF OUR GRANT WRITING EFFORTS
TO AND I CREATED PHOENIX NEST
AFTER WATCHING THE TRIALS AND
THE TRIBULATIONS THAT THE SAM A
AND SAM B GROUPS WERE GOING
THROUGH WITH THEIR TREATMENTS
THEY HAD GONE TO THE CLINIC AND
JUST REALLY HAD NOT FOUND THE
CORRECT END POINTS AND GOTTEN
ACROSS THE FINISH LINE.
SO, I REALIZED THAT BECAUSE OF
THE RARITY OF S SANFILIPPO WE HA
SPONSOR PICK US UP AND I CO
FOUNDED PHOENIX NEST IN 2012 TO
HELP BRING THE TREATMENTS THAT
ARE NON PROFITS FUNDRAISED TO
THE CLINIC.
WE HAVE THREE DRUG PROGRAMS AND
TWO NATURAL HISTORY STUDIES IN
OUR TYPE LINE.
WHAT WE CAN'T DO IN-HOUSE WE USE
THE EXPERTISE OF OUR CONSULT
APARTCONSULTANTSAND CONTRACTORS.
SO SOME OF THE PROS AND CONS OF
WRITING SBR GRANTS ARE THE
PRIMARY FOCUS OF THE NIH GRANTS
THAT WE WRITE GRANTS FOR AND A
EMPLOYING FOR GRANTS IS
EXTREMELY ARDUOUS AND
HEARTBREAKING.
YOU PUT SO MUCH INTO WRITING
THESE GRANTS AND IT TACKS A LONG
TIME.
AND SOMETIMES YOU DON'T WIN.
ACTUALLY A LOT OF TIMES YOU
DON'T WIN.
IT'S HEARTBREAKING.
YOU CAN COME INTO A LOT MORE
PROBLEMS TOO WITH IF YOU ARE
FUNDING IS FROZEN AND YOU DIDN'T
MAKE A MILESTONE AND IF THERE'S
A GOVERNMENT SHUT DOWN, THERE'S
ALL KINDS OF THINGS THAT CAN
HAPPEN TO YOU ON THIS ROAD OF
BEING FUNDED BY NIH GRANTS BUT
FOR THOSE OF YOU WHO KNOW HOW
HARD IT IS TO DO GRASSROOTS
FUNDRAISING I TAKE THE WRITING
THE GRANTS ANY DAY.
ALSO, IN ORDER TO GET YOU TO THE
POINT WHERE YOU CAN WRITE OR WIN
A GRANT, YOU MUST GO THROUGH
THIS LONG LIST OF LONG-TO DO
LIST OF APPLYING AND REGISTERING
FOR ALL THESE INSTITUTIONS.
AND EACH REGISTRATION IS
CONTINGENT ON THE INSTITUTION
ACCEPT YOU IN THE PROCEEDING
LIST OF THINGS TO DO IF THAT
MAKES SENSE.
THEY ALL FLOW TOGETHER.
YOU CAN'T HAVE ONE WITHOUT
ANOTHER AND IT TAKES A REALLY
LONG TIME TO GO THROUGH ALL OF
THIS.
GIVE YOURSELF FOUR TO SIX
MONTHS.
THIS IS OUR GRANT HISTORY AND WE
GO FOR THE SBIR GRANTS AND WHICH
SIDE AM I ON?
THEY HAVE LONG COMPLICATED
NUMBERS HERE WHICH REPRESENT THE
TYPE OF GRANT THAT YOU'VE GONE
FOR.
AND ALMOST IN CLOSING HERE, HERE
IS MY FAVORITE SLIDE.
I KNOW IT'S SUPER BUSY HERE BUT
HERE IS AN OUTCOME OF A GRANT
WE'RE WORKING ON RIGHT NOW AND
CALLED FROM THE
COMMERCIALIZATION READINESS
PILOT PROGRAM GRANT WHICH I HOPE
IS NOT GOING TO JUST BE A PILOT
THAT IT WILL BE AROUND TO STAY.
THIS IS THE THIRD IN A SERIES OF
GRANTS AND THIS GRANT IS FOCUSED
ON A NATURAL HISTORY STUDY FOR
TYPE D, I THINK IN OUR LAST
CONVERSATION WE HAD A LOT OF
GOING ON AROUND NATURAL HISTORY
STUDIES AND THE PORT APPS OF
NATURAL HISTORY STUDIES AND I
CAN'T ECHO THIS ENOUGH,
ESPECIALLY FOR THE TALL RARE ULE
DISEASES WHERE THERE'S NOTHING.
IN THE SPACE OF SANFILIPPO AND I
CANNOT WAIT TO KICK IT OFF AND
SEE THIS GO LIVE AND IN ACTION.
THERE'S -- WE ARE THE LAST TYPE
C AND D ARE THE LAST TO BE GOING
TO THE CLIP I CAN HERE AND THAT
IS KIND OF A SILVER LINING WHEN
YOU HAVE ARE THE LAST, YOU GET
TO SEE WHERE THE OTHER SPONSORS
MAY HAVE NOT NECESSARILY MESSED
UP BECAUSE YOU NEVER KNOW WHEN
YOU START THERE BUT GAPS.
THIS NATURAL HISTORY STUDY WILL
FILL IN THE GAPS.
WE HAVE DESIGNED AN APP WHICH IS
EXACTLY WHAT IT SOUNDS LIKE.
AN APP YOU CAN DOWN LOAD ON TO
YOUR PHONE AND THE FAMILY WILL
BE GIVEN SEVERAL TASKS THAT ARE
SANFILIPPO-RELATED AND THESE
TASKS WILL ECHO WHAT IS
HAPPENING IN THE CLINIC BUT IN
THE CLINIC, YOU DON'T GET A GOOD
SENSE OF THE CHILDREN, WHERE THE
KIDS DO NOT COOPERATE AND THEY
WALK INTO THE CLINIC AND THEY
RUN FOR THE HILLS, THEY'RE OUT
THE DOOR.
THAT'S THE KIDS THAT CAN RUN.
WE'RE GOING TO HAVE FAMILIES GO
THROUGH AND DO IN A VERY
STANDARDIZED FORM SAT,
CONTROLLED FORMAT, GO THROUGH
AND VIDEOTAPE SEVERAL ACTIVITIES
THAT REVOLVE DAILY LIVING
ACTIVITIES AND WE ARE ALSO
DESIGNING OUR NEW CLINICAL
OUTCOME ASSESSMENTS AND I DON'T
KNOW IF WE'VE TALKED ABOUT
CLINICAL OUTCOME ASSESSMENTS IF
PEOPLE ARE AWARE OF WHAT CLIP
CAL OUTCOME ASSESSMENTS ARE BUT
IF YOU ARE STARTING OUT, I
REALLY ENCOURAGE YOU TO GET A
GOOD FEEL OF WH WHAT THESE
MEASUREMENT RIGHT SIDE AND WHAT
IS APPROPRIATE FOR OUR PATIENT
POPULATION.
I SEE ON THE CHAT PEOPLE ARE
ASKING ABOUT END POINTS AND THE
IMPORTANCE OF END POINTS AND
YEAH I CAN'T BEHIND THAT ENOUGH,
YOU ARE ON THE RIGHT TRACK
ASKING THOSE QUESTIONS AND IT'S
PERFECT TIMING THE FDA JUST CAME
OUT WITH A DRAFT GUIDANCE FOR
PATIENT-FOCUSED DRUG DEVELOPMENT
AND SELECTING AND DEVELOPING
CLINICAL OUTCOME ASSESSMENTS SO
THIS IS TIMELY FOR THIS
CONVERSATION AND I FEEL LIKE I
JUST SPED THROUGH 12 YEARS OF MY
LIFE IN EIGHT MINUTES AND I HOPE
IT WAS HELPFUL.
THE FAMILIES ARE THE FUEL THAT
KEEPS ME GOING AND TO OUR
WONDERFUL PROGRAM OFFICERS JILL
AND EMILY WHO YOU WILL MEET
NEXT.
THANK YOU AND I LOOK FORWARD TO
THE QUESTIONS AND THE ANSWERS.
>> IT'S AMAZING THAT YOU HAVE
BEEN ABLE TO ACCOMPLISH IT AND
IT'S CREDIBLE YOU HAVE ADDED
$10 MILLION OF FUND TO GO
SANFILIPPO RESEARCH AND
SUFFICIENT THIS CLINICAL
OUTCOMES CEASEMENT PROJECT FOR
NERLY THREE AND A HALF MILLION
DOLLARS AND IT'S INCREDIBLE.
AS JILL MENTIONED, THE WORK THAT
THEY'VE BEEN ABLE TO ACCOMPLISH
HAS BEEN FUNDED THROUGH THE
NINDS RESEARCH TRANSFER PROGRAM
AND THE PROGRAM DIRECTOR IN THE
DIVISION OF TRANSLATIONAL
RESEARCH AT NINDS IS Dr. EMILY
AND SHE JOINED FROM INDUSTRY
BYWAY OF THE DEPARTMENT OF
DEFENSE AND SHE'S A FOUND I
REMEMBER OF A SMALL BUSINESS
HERSELF SO SHE WILL TALK ABOUT
THE NIH SMALL BUSINESS PROGRAMS
WHICH HAPPEN TO BE THE LARGEST
SOURCE OF SPECIFICALLY TO
SUPPORT SMALL BUSINESS RESEARCH
AND DEVELOPMENT.
>> THANK YOU SO MUCH.
I THINK I HAVE CONTROL OVER THE
SLIDES AND I'LL WALK THROUGH AND
IT'S A PLEASURE TO FOLLOW OUR
PROGRAM THAT IS VERY PROUD OF
THE SUPPORTING HER AND HER
EFFORTS AND REALLY I THINK SHE
HAS BEEN SO SUCCESSFUL WITH
SMALL BUSINESS FUNDING AND SO
TODAY I'M GOING TO TALK A LITTLE
BIT ABOUT WHAT IS IT LIKE TO
APPLY FOR BRAVE SOULS WHO WANT
TO FOLLOW JILL DOWN THE PATH OF
STARTING A SMALL BUSINESS OF
THERAPEUTIC DEVELOPMENT AND
EDUCATE THE COMMUNITY, THE
NON-PROFIT COMMUNITY ABOUT WHAT
SMALL BUSINESS FUNDING GRANTS
ARE LIKE WHAT'S OUT THERE AND
SOME WAYS THAT NON PROFITS CAN
HELP THESE EARLY STABLE SMALL
BUSINESSES AND ACHIEVE THEIR
GOALS.
OK, LET'S DO IT.
SO WHAT IS SBIR AND STTR THIS
ACRONYM WE'RE REFERRING TO THIS
REFERS TO CONGRESSIONAL REVIEW
DATED SMALL BUSINESS PROGRAMS
AND THE SMALL BUSINESS
INNOVATION RESEARCH PROGRAMS AND
THE SMALL BUSINESS TECHNOLOGY
TRANSFER PROGRAM.
BOTH OF THESE PROGRAMS REQUIRED
THAT FEDERAL FUNDERS LIKE NIH
SEND THEIR FUNDING DOLLARS ON
SMALL BUSINESSES AND THERE ARE
SOME SMALL DIS TANKION BETWEEN
WHAT MAKES AN SBIR VERSUS AN
STTR IT HAS TO DO WITH
PARTNERING WITH THE UNIVERSITY
AND I'M NOT GOING TO DO INTO IT
TODAY AND IT'S NOT RELEVANT TO
UNDERSTAND THE BASICS BUT IF YOU
ARE INTERESTED IN A EMPLOYING
FOR THIS FUNDING I'LL WALK
THROUGH WHICH MECHANISMS MAKE
THE MOST SENSE.
THE PORTAGE THING TO UNDERSTAND
THESE ARE GRANTS AND THEY'RE NOT
LOANS SO YOU DO NOT NEED TO PAY
THIS MONEY BACK.
IT'S GRANT FUNDING SO YOU ARE
NOT LOSING ANY PERCENT OF
OWNERSHIP OR EQUITY IN YOUR
COMPANY AND SMALL BUSINESS
AWARDS, ESPECIALLY I WITH SAY
NIH AWARDS WHICH GO THROUGH IT
RIGOROUS PEER REVIEW PROCESS ARE
RECOGNIZED AS BEING A GOLD STAR
ON YOUR COMPANY AND IT CAN BE
USED TO HELP LEVERAGE ADDITIONAL
WE SUPPORT THE VERY EARLY STAGE
OF RESEARCH TYPICALLY MAYBE RYE
AFTER A LICENSING AGREEMENT
HAPPENED WITH THE UNIVERSITY OR
A A VERY EARLY PROTOTYPING STAGE
AND WE'LL FUND THROUGH EARLY
STAGE CLINICAL TRAILS AND REALLY
OUR GOAL IS TO HELP DERISK THAT
TECHNOLOGY AND TO A POINT WHERE
IT'S EITHER READY FOR PRIVATE
FUNDING AND MUCH MORE LARGER
DOLLAR AMOUNT THAN WE'RE ABLE TO
PROVIDE FOR GRANT FUNDING OR FOR
SOME TECHNOLOGIES LIKE AS SAY OR
RESEARCH TOOLS THEY MAY GET TO
THAT POINT OF BEING COMMERCIAL
READY THROUGH GRANT FUNDING SO
WHAT DOES IT TAKE TO BE ELIGIBLE
FOR SBIR AND STTR FUNDING, WE
HAVE TO BE ABLE FORMED AND
REGISTERED U.S.-BASED SMALL
BUSINESS AND IT HAS TO BE A FOR
PROFIT BUSINESS AND IT'S WISE TO
START THOSE FOUR MONTHS BEFORE
YOU PLAN ON APPLYING BECAUSE YOU
ARE BUSINESS HAS TO BE
INCORPORATE AND REGISTERED
BEFORE YOU APPLY.
THE COMPANY NEEDS TO BE LESS
THAN 500 PLOY SKIS THIS CRITERIA
IS NOT HARD THE COMPANIES ARE
MAYBE FIVE OR SO PEOPLE AND IT'S
PROBABLY ABOUT AVERAGE AND IT
NEEDS TO BE U.S. INDIVIDUAL
OWNED.
MULTIPLE U.S. INDIVIDUALS AND IT
NEEDS TO BE MAJORITY OWNED BY
U.S. INDIVIDUALS.
THERE'S A LOT OF LEGAL EASE
BEHIND THIS DEFINITION AND I'M
NOT GOING INTO THIS TIME BUT
THIS INFORMATION IS PUBLIC AND
I'M HAPPY TO WALK THROUGH ANY
QUESTIONS FUR NOT QUITE SURE
WHETHER YOU ARE -- IT'S GOING TO
BE UP TO THE BUSINESS TO CERTIFY
THAT YOU ARE ELIGIBLE FOR SMALL
BUSINESS FUNDING AT THE TIME
THAW RECEIVE YOUR AWARD.
SO AT NINDS THE AMOUNT OF
FUNDING DEDICATE TODAY SMALL
BUSINESS IS OVER $80 MILLION A
YEAR AND IT SUPPORTS ALL OF THE
INDICATIONS WITHIN THE MISSION
SPACE AND SO THAT IT INCLUDES
LARGE INDICATIONS LIKE STROKE
AND ALSO MANY OF THE RARE
DISORDERS THAT ARE REPRESENTED
HERE TODAY AND WHICH PHOENIX
NEST IS WORKING TO SUPPORT.
WE HAVE MODALITIES OF
TECHNOLOGIES SO ABOUT PROBABLY
OVER HALF OF OUR PORTFOLIO IS
SMALL MOLECULES OR ANNA RARE OF
DIFFERENT BIOLOGICAL APPROACHES
TO GENE THERAPY, THEY WILL
THERAPY AND ET CETERA A REALLY
BROAD SCOPE THERE BUT WE FUND
DIAGNOSTICS, RESEARCH TOOLS,
BIOMARKERS, DEVICES, ANYTHING
THAT WILL MAKE ITS WAY THROUGH
THE COMMERCIALIZATION PATH TO BE
A PUBLIC THAT WILL HELP BENEFIT
THE PATIENTS AND RESEARCH
COMMUNITIES THAT NINDS SUPPORTS.
THIS STAGE OF RESEARCH CAN BE
BROAD AS WELL SO WE SUPPORT THE
VERY EARLY STAGE WORK IN THE
TRANSLATION ALDO MAIN THROUGH
IND TO BE READY FOR CLINICAL
TRIALS IN THE FUND PHASE 1 AND
EVEN PHASE 2 CLINICAL TRIALS
UNDER OUR FUNDING.
>> THERE'S A NEED TO DO FOREIGN
BASED WORK AND IT CAN'T BE UNDER
THE CLASS OF A SMALL BUSINESS OR
SBIR AWARD.
IT'S A FEE THAT GOES TO THE
COMPANY AND IT CAN BE USED
HOWEVER YOU LIKE BUT IT'S
LIMITED AND I MENTIONED THIS
BECAUSE ONE WAY WE FOUND THAT
SMALL BUSINESSES REALLY
STRUGGLING TO DEVELOP THEIR
THERAPEUTICS IN THE EARLY STAGES
CAN BENEFIT FROM ENGAGEMENT WITH
NON PROFITS IS SMALL AMOUNTS OF
FUNDING THE COMPANIES CAN BE BUT
NOT GETTING THEIR PATENT FEES OR
THEIR LICENSE FEES OR THEIR
OTHER EXPENSES AND THESE ARE
ESSENTIALS TO SECURING THE IP
AND FOR THEIR TECHNOLOGY BUT
THEY REALLY STRUGGLED TO FIND
THAT FUNDING SO I'LL JUST
MENTION THAT.
AFTER YOU SUBMIT THAT
APPLICATION IN AND AS JOE
MENTIONED I'M NOT GOING TO SHY
AWAY FROM IT IT'S AN OWNER US
APPLICATION THEY'RE 90 TO 100
PAGES AND THEY'RE INTENTION AND
AFTER YOU SUBMIT THAT IT'S GOING
TO GO THROUGH PEER REVIEW AND
THAT PEER REVIEW IS SIMILAR TO
NIH TYPICAL NIH RESEARCH
ACADEMIC RESEARCH PER REVIEW AND
THESE PANELS DO INCLUDE MEMBERS
OF INDUSTRY AND CLINICIANS AND
OTHER PEOPLE WITH TRANSLATION
ALEX PER TEASE.
THE STANDARD THINGS HAVE YOU ON
ANY RESEARCH ARE EVALUATED WITH
THE ADDITIONAL AN ALLEY
EVALUATION OF THE
COMMERCIALIZATION POTENTIAL.
PRODUCT AND I WANT TO STRESS
HERE THAT IT'S A COMPETITIVE
PROGRAM AND FUNDING PERCENTAGES
HAVE HOVERED BETWEEN 15% TO 20%
AND I WANT TO ACKNOWLEDGE THAT A
LOT OF TIMES YOU ARE COMPETING
AGAINST SMALL BUT ESTABLISHED
BIOTECH COMPANIES AND SO
BRIDGING IN A TEAM AND YOU WANT
TO SHOW THAT YOU ARE A CAPABLE
COMPANY AND YOU CAN TAKE THIS
FORWARD TO THE POINT WHERE IT
COULD BE FURTHER WHERE GROUPS
CAN REALLY THOUGH SUPPORT SMALL
BUSINESSES IS PROVIDING THOSE
LETTERS OF SUPPORT AND IF YOU
CAN PROVIDE LETTERS OF SUPPORT
FROM PATIENTS AND FAMILIES
THEMSELVES, FROM CLINICIANS AND
WHO WORKED WITH AND OR IF YOU
HAVE EARL STAGE CONVERSATIONS
WITH PARTNERS HAVING THAT
SUPPORT OF THEM, IT DOESN'T HAVE
TO BE A COMMITMENT BUT STATING
THEIR INTEREST SHOWS THAT
COMMERCIALIZATION POTENTIAL OF
YOUR TECHNOLOGY.
AND ANOTHER ONE IN THIS ONE IS
REALLY HARD AS JILL MENTIONED
IT'S NOT UNCOMMON FOR
APPLICATIONS NOT TO BE PURCHASED
ON THEIR FIRST TRY YOU WILL GET
A LIST OF COMMENTS WE'LL GET A
LOT OF FEEDBACK AND I OR YOUR
PROGRAM OFFICER WE CAN GO
THROUGH HOW WE CAN STRENGTHEN
THE PROPOSE AM AND IMPROVE THIS
TO GET TO A FUNDABLE RANGE.
I ALSO WANT TO NOTE OUR PROGRAM
DOES HAVE ADDITIONAL SUPPORT
PROGRAMS FOR NEW APPLICANTS
SPECIFICALLY AND THE APPLICANT
ASSISTANCE PROGRAM IS INTENDED
TO PROVIDE GRANT-WRITING SUPPORT
FOR A PHASE 1SBIR TO ANY COMPANY
WHO HAS NEVER BEFORE RECEIVED
NIH, SBIR OR STTR FUNDING AND IN
BEING, WE HIGHLY ENCOURAGE
DIVERSE APPLICATIONS TO A PLOY
TO THIS PROGRAM DIVERSITY AND
SMALL BUSINESS INCLUDES
WOMEN-LED COMPANIES.
IF YOU ARE INTRODUCED IN
PURSUING SMALL BUSINESS FUNDING
AND STARTING YOUR COMPANY AND
PURSUING THIS PATH WOULD BE TO
REACH OUT TO OUR TEAM IT'S
HELPFUL TO HAVE A DESCRIPTION OF
THE PROJECT YOU ARE THINKING
ABOUT EVEN IF IT'S A DRAFT OR
NOT CLEAR SO WE CAN SORT OF GET
A SENSE OF WHERE YOUR READINESS
IS AND WHAT WE SHOULD RECOMMEND
BUT WE'RE HAPPY TO TALK WITH YOU
AND OFTEN TIMES A LOT OF THE
QUESTIONS ARE VERY SPECIFIC SO
IF IT'S BEST TO JUST GET ON A
CUL AND TALK THROUGH THAT.
I'M GOING TO PAUSE THERE AND I'M
HAPPY TO ANSWER ANY QUESTIONS.
>> THANK YOU, EMILY.
THAT WAS REALLY HELPFUL
OVERVIEW.
OUR NEXT SPEAKER IS KEN FROM
SHANEER AND LUBITZ.
IT'S A FIRM THAT REPRESENTS TAX
INCENTIVE ORGANIZATIONS.
WHETHER YOU ARE A PATIENT
ADVOCACY GROUP CONTEMPLATING
STARTING A COMPANY OR YOU ARE
THINKING OF WORKING WITH AN
ACADEMIC CENTER OR EVEN JUST
THINKING ABOUT LIKE THE ABILITY
TO SUB LICENSE RESOURCES OR AS
SAYS YOU HELPED TO FUND LIKE
THEY WERE DISCUSS NORTH THE
PREVIOUS PANEL, YOU WILL WANT TO
BE MINDFUL ABOUT PROJECTING YOUR
501C3 STATUS AND SO KEN WORKS
WITH GROUPS AND HE HAS SERVED AS
COUNCIL TO CYSTIC FIBROSIS IN
THEIR TRANSACTION WITH -- HE IS
OUTSIDE COUNCIL TO NON-PROFIT
GROUPS ADVISING ON A WIDE RANGE
OF ISSUES.
ARE YOU READY?
>> YOU ARE MUTED.
>> ALL RIGHT, I'M UNMUTED.
MAYBE I WOULD BE MORE
INTERESTING MUTED.
WHO KNOWS.
THANK YOU FOR HAVING ME.
I REPRESENT ABOUT 80 DIFFERENT
DISEASE RELATED CHARITIES AND
CYSTIC FIBROSIS IS ONE OF THEM,
MICHAEL J. FOX -- A BUNCH.
SOME BIG AND SOME SMALL AND MOST
GIVE AWARDS TO COMPANIES TO
DEVELOP PRODUCTS SO THE PURPOSE
TODAY IS TO TALK ABOUT WHAT KIND
OF AGREEMENTS THAT THEY, THE
CHARITIES EXPECT FROM THE
APPLICANTS AND WHAT FORMS THEY
WILL TAKE SO LET ME SAY I'VE
DONE THE CYSTIC FOUNDATION AND
EVERYONE TALKS ABOUT IT AS THE
MOTHER OF ALL VENTURE
PHILANTHROPY AND WHEN
TRANSACTIONS THAT WAS FIRST
PIECE WAS IN 2002 AND THE
COMPANY CALLED BIO SCIENCE AND
THEY MADE THE FIRST AND WE DID
SUPPLEMENTAL FUNDING WITH VER
TEXT THAT ADDED UP TO
$125 MILLION OR SO AND
ULTIMATELY IT'S ALL PRODUCTS AND
CYSTIC FIBROSIS FOUNDATION SOLD
IT'S ROYALTY INTEREST UNDER
THOSE AGREEMENTS AND REAPED
ABOUT FOUR AND A HALF BILLION
DOLLARS OF FUNDING, REALLY AN
EXCITING AND SHOULD HAPPEN TO
ALL OF US-TYPE EXPERIENCE AND
ULTIMATELY THAT MONEY WILL ALLOW
THEM TO CURE THE DISEASE.
AND I THINK I PROBABLY HAVE DONE
FIVE TO 600 OF THESE
TRANSACTIONS AND ONE AND MAYBE
THE TOTAL IN MAYBE COMING UP
WITH A BILLION DOLLARS OF
FUNDING WITH YOU DON'T GET
SCARED BECAUSE A LOT OF THEM ARE
SMALL, SMALL START UP COMPANIES
AND THAT'S WHAT I WANT TO FOCUS
ON TODAY BECAUSE THE FUNDING IN
THOSE TRANSACTIONS CAN GO FROM
100,000 TO A MILLION AND I
WANTED TO TALK ABOUT THE KINDS
OF AGREEMENTS THAT YOU WILL
ENTER INTO.
SO LET ME START WITH THE FIRST
ONE.
AT THE BEGINNING, LEADING THE
PACK, THE THOUGHT IT COULD BE A
CONFLICT 20 INVEST IN STOCK AND
EQUITY.
SO MOST OF THE EARLY
TRANSACTIONS AND A BUNCH OF THEM
STILL ARE BASED ON ROYALTIES AND
THE ROYALTIES FOR SMALL
TRANSACTIONS ARE USUALLY NOT A
PERCENTAGE OF NET SALES BUT THEY
ARE A MULTIPLE OF THE AWARD AND
SO IF AT WARD IS $100,000 AND
THE DRUG A APPROVED, THE CHARITY
WOULD GET BACK THREE TO 6X
CALLED ROYALTY OR UPON FIRST
COMMERCIAL SALE OF THE PRODUCT
AND N ARE POTENTIAL MILESTONES
WHERE THE CHARITY PARTICIPATES
WITH THE COMPANY IN SUCCESS AT
CERTAIN LEVELS SO IF AGGREGATE
SALES REACH WHAT IS DEFINED IN
THE AGREEMENTS AS A SUCCESS
LEVEL, THEN THE CHARITY GETS AN
EXTRA ONE TO TWO SO THOSE ARE
NICE TO THINK ABOUT.
NICE TO THINK ABOUT APPROVALS
AND MOST OF TIME ON THESE
TRANSACTIONS THE PAYMENTS OCCUR
AT EXITS.
THAT IS WHEN THE SMALL COMPANY
SELLS ITSELF AND LICENSE IS THE
PRODUCT SELLS ITSELF TO A LARGER
PHARMA THAT HAS THE A LOT TO
TAKE A PRODUCT TO MARKET.
SO THE WAY THAT WORKS IS THE
CHARITY IN SUCH CASES GETS A
PERCENTAGE OF THE CONSIDERATION
RECEIVED BY THE COMPANY UP TO
THE ROYALTY CAP WHICH WE TALKED
ABOUT WHICH WAS THE GREATEST
SUCCESS.
AND THE CHARITY IS THEN PAID OFF
ITS AMOUNT AND THE COMPANY
HOPEFULLY TAKES THE PRODUCT TO
MARKET.
AND I THINK THE PRIOR PANEL I
SAT IN FOR THE TAIL END OF THE
PRIOR PANEL, I ALSO INCLUDE WHAT
I CALL AN INTERRUPTION LICENSE.
THERE'S ALWAYS THE WORRY AND
I'LL TELL YOU THE ONE DAY A
LONG, LONG TIME AGO NOW, AFTER
THE FIRST VER TEXT AND SO WHAT I
PUT INTO ALL THESE AGREEMENTS
NOW, THE COMPANY STOPS WORKING
AND A PARTICULAR OF THE FUND
THAT COMPOUND OR DIAGNOSTIC OR
DEVICE AND THE LICENSE OCCURS
AND THE COMPANY AND THE CHARITY
TAKES OVER AND BRINGS IT TO
MARKET.
FROM A COMPANY'S STANDPOINT, ONE
CAN, THERE CAN BE A LOT OF
REASONS WHY THE COMPANY STOPS.
THEY RUN OUT OF MONEY, ET
CETERA.
AND I THINK THE CHARITIES ARE
SAYING IF THIS HAPPENS TO YOU,
WE'LL SHARE REVENUE WITH REVENUE
WITH YOU AT A CERTAIN SELF AND
ROYALTIES I WOULD SAY OF THE 15
YEARS OR SO AND THEY'VE BEEN
DOING THIS 20 YEARS, 20 YEARS.
AND ROYALTY BASED AGREEMENTS
WERE BUY AND LARGE THE MAJORITY
OF THE FIRST 10 YEARS OR SO AND
THEN CHARITIES THEY CAN BE
DIRECT EQUITY.
THIS SIDE IS OUT OF ORDER BUT
THEY CAN BE DIRECTLY INEQUITY
AND I'M GOING TO SWITCH TO --
THEY'RE OUT OF ORDER SO I'M
GOING TO SWITCH TO THE NEXT ONE.
IT ANTICIPATES A LARGER FUNDING
FOR THROW YEARS AND THEY TAKE
THE TAKE THE FORM OF A SAFE
WHICH IS THE SIMPLE AGREEMENT
FOR EQUITY OR CONVERTIBLE AND
WHEN THE COMPANY DOES ITS LARGER
FUNDING AND THE CONVERSION RATE
IS AT A 10% TO 30% DISCOUNT OVER
WHAT THE LARGER FUNDING PEOPLE
WHO PARTICIPATED IN THE LARGER
FUNDING PAY AND THE MAJOR
DIFFERENCE BETWEEN A SAFE AND
CONVERTIBLE NOTICE, THE
CONVERTIBLE NOTE BEARS INTEREST
USUALLY AT THE SIX TO 12%
DEPENDING UPON THE NEGOTIATIONS
AND THE SAFE DOES NOT I'M NOT
CRAZY ABOUT SAVES FOR THAT
REASON BECAUSE IT'S UNFAIR, I
THINK, FOR THE CHARITIES THAT
HAVE THE MONEY OUT THERE WITHOUT
HAVING AN INTEREST ON THEM.
SO, EQUITY AGREEMENTS THAT
RELATE TO OFTEN HAVE A SIDE LIST
SO THE CHARITIES ARE INVESTING
IN A COMPANY THAT MAY HAVE MORE
THAN ONE PARTICULAR PRODUCT AND
WHILE THE CHARITY IS ONLY
FOCUSING ON A PRODUCT THAT
RELATES TO ITS DISEASE SO THE
SIDE AGREEMENT REQUIRES THAT THE
MONEY THAT THE CHARITY PUTS IN
IS FOCUSED AND USED PRIMARILY
FOR THE PARTICULAR DISEASE SO
FOR EXAMPLE, IF I'M REPRESENTING
NATIONAL KIDNEY WHICH IS A
REALLY GOOD GROUP THAT DOES
EQUITY FUNDING AND LET'S SAY IT
INVESTS UP $200,000, IT WOULD
REQUIRE THAT THE COMPANY USE ITS
MONEY SOLELY FOR THE PARTICULAR
THERAPY THAT THE NATIONAL IS
FOCUSING ONMENT A COUPLE OTHER
REASONS YOU WOULD FOCUS ON USE
OF SIDE AGREEMENT WOULD BE MOST
FAVORED NATION AGREEMENT SAYS
THE CHARITY IS GETTING THE BEST
DEALS AND SOMETIMES THE CHARITY
LIKES AN OBSERVER SEAT ON THE
BOARD OF THE DIRECTORS AND THE
SIDE AGREEMENT REQUIRES THAT AND
IF NEITHER OF THOSE POSSIBLE,
THE CHARITY REQUIRES REPORTING
AND IT'S PARTICULAR BECAUSE
THEY'RE TAKING A RISK AND
INVESTING AT THE EARLIEST STAGES
AND THEY MAY WISH TO INVEST
SUBSEQUENTLY AND YOU MAY WISH TO
HAVE THEM INVEST SUBSEQUENTLY.
SO I'M GOING TO SWITCH TO THE
LAST SLIDE AND SO SOMETIMES THE
CHARITY IS COMING IN AT A
FUNDING ROUTE AND SO THERE WON'T
BE THIS INTERVENING SAVOR A
CONVERTIBLE NOTE THEY'LL INVEST
DIRECTLY AND THERE WILL BE
SHAREHOLDER AGREEMENTS THAT YOU
THE COMPANY WILL BE ASKED TO
PREPARE FOR THE CHARITY DOCUMENT
REGISTRATION RIGHTS AND SALES
AND TRANSFER RESTRICTIONS AND ET
CETERA, ET CETERA.
AND INCLUDING RIGHT TO
PARTICIPATE IN FUTURE ROUNDS.
AND SO THESE TOO ARE OFTEN THE
CHARITY OFTEN REQUIRES THE SIDE
AGREEMENT TO MAKE SURE THAT IT'S
FUNDING WAS USED FOR THE TARGET
PRODUCT.
SO THAT'S ALL.
THERE'S BEEN A COUPLE QUIZ ABOUT
THE SLIDES.
THEY'RE AVAILABLE AND I'M HAPPY
TO SMALL COMPANIES WISH TO CALL
ME TO CHAT WITH THEM ABOUT WHAT
THEIR AGREEMENTS SHOULD SAY.
SAY JUST WANT TO SAY I SPENT A
35 YEAR CAREER AND BILLING LAW
AND REPRESENTING COMPANIES AND
THAT WAS FINE AND I ENJOYED IT
AND STARTED A FIRM WITH A FEW
FRIENDS THAT GROUP TO 300 AND ET
CETERA BUT THIS LAST 10 YEARS OF
WORKING WITH DATA AND CHARITIES
AND FUNDING THERAPIES AND
DEVICES IN DIAGNOSTICS FOR
DISEASE CHARITIES HAVE BEEN THE
BEST 10 YEARS OF MY PRACTICE.
THE DAY WE FOUND OUT THAT THE
FIRST CF DRUG WORKED AND GOING
TO MARKET, I'M NOT A SCIENTIST
SO I COULDN'T SAY I HAD CREDIT
FOR IT BUT IT WAS A GREAT POINT
IN MY CAREER.
SO THANK YO FOR HAVING ME.
>> THANK YOU SO MUCH.
WE HAVE A FEW MINUTES WE CAN
TAKE SOME QUESTIONS RIGHT NOW IF
ANYBODY HAS ANY QUESTIONS YOU
ARE WELCOME TO JUST TYPE THEM
INTO THE CHAT.
>> I CAN BE REACHED AT THE
WEBSITE.
>> THANK YOU.
>> KEN, HOW OFTEN DO YOU SEE A
SITUATION SORT OF LIKE PHOENIX
NEST?
WHERE A PATIENT ADD CO KIT SAYS
I'M GOING TO START A COMPANY.
>> I SEE IT FAIRLY OFTEN AND SO
WE DON'T -- WE GENERALLY
REPRESENT THE FOUNDATION NOT THE
COMPANY.
WE GET INVOLVED SOMETIMES
THERE'S STILL ACADEMIC GRANTS.
THAT GO WAY BACK TO THE EARLY
2000s AND THE FIRST QUESTION
CYSTIC FIBROSIS ASKED ME, NOT
THE FIRST QUESTION BECAUSE I
REPRESENTED YEARS BEFORE BUT ONE
OF THE KEY QUESTIONS WAS COULD
IT FUND INDUSTRY?
COULD IT FUND THE COMPANY?
AND UP TO THEN, MOST FOUNDATIONS
WERE THOUGHT THEY COULD ONLY
MAKE ACADEMIC GRANTS.
THEY DIDN'T THINK THEY COULD
FUND COMPANIES AND SO WE GOT
PAST THAT ISSUE EARLY ON AND SEE
HOW IT STARTED FUNDING COMPANIES
AND NOW SOMEONE CAME UP WITH THE
MAIN WHICH WAS QUITE CLEVER AND
IT'S BECOME APP AREA THAT THE
FOUNDATIONS HAVE FOCUSED ON.
>> WE HAVE A COUPLE QUESTIONS.
HOW LARGE DOES A CHARITY NEED TO
BE TO MAKE THESE TYPES OF
ATTEMPTS FEASIBLE?
>> REALLY SMALL.
I DON'T WANT TO PRETEND THAT
WITH THE FUNDING WAS
$25 MILLION.
THERE'S NOT GOING TO BE MANY OF
THOSE AND THE MILLION DOLLARS OR
SO OF FUNDING MOST OF THEM IF
YOU TOOK AWAY THE BIG BIG
AMOUNTS, EVEN PFIZER AND FUND
LILLY, ET CETERA AND THE REASON
FOR THAT IS THERE'S A GUY WOULD
WORKS FOR THEM AND WHO WANTS TO
GO TO HIS WINDOWS AND SAY I HAVE
THIS IDEA FOR A PROGRAM AND I
CAN GET FUNDING FOR A CHEETAH
THAT HELPS HIM JUMP START HIS
OWN PROJECT AND MOST OF THE
THESE FUNDINGS TAKE NATIONAL
KIDNEY WHICH I'VE BEEN DOING SIX
OR SEVEN DEALS FOR IT AND THOSE
ARE BETWEEN 100,000 AND 250 AND
I'LL DONE DEALS AS LOW AS 50,000
AND LET ME JUST SAY I ENJOYS
THOSE JUST AS MUCH AND I HOPE
THEY'RE SUCCESSFUL AND YOU KNOW,
JILL'S STORY WAS FABULOUS.
THERE ARE SMALLER DEALS WITH
SMALL START UP COMPANIES THAT
WORK FOR BOTH PARTS.
>> ARE THERE ANY TYPES OF
ININDUCE TREMENDOUS INVESTMENTS
THAT YOU DO NOT RECOMMEND?
WHO ORGANIZATIONS ENGAGE WITH?
>>
>> WELL, I DON'T THINK I --
OBVIOUSLY, FROM THE CHARITY
STANDPOINT, THEY'RE LOOKING FOR
THERAPY OR DIAGNOSTIC OR ADVICE
THAT IS GREAT FOR THE PATIENTS
THAT IS NOT AVAILABLE AND YOU
ARE OBVIOUSLY LOOKING FOR A
COMPANY THAT'S NOT GOING TO GO
BROKE USE YOUR MONEY AND THEN
GOT TO GO GET WHATEVER PROGRESS
WILL BE LOST BY THE FACT THEY GO
BANKRUPT SO SOMETIMES WHEN WE
LOOK AT THE BALANCE SHEET
THEY'RE THIN AND THEY'RE THIN SO
I WOULD SAY IT'S MAINLY WITH
PEOPLE WHO HAVE A GOOD IDEA WHO
HAVE SOME EXPERIENCE AND
CREDIBILITY AND ALL KINDS OF
THINGS AND.
>> ARE THERE ANY RESOURCES FOR
PATIENT AT VOW KIDS OR ACADEMIC
INVESTIGATORS CAN LEARN MORE
ABOUT A EMPLOYING FOR SMALL
BUSINESS GRANTS?
>> YEAH, ABSOLUTELY SO I CAN PUT
INTO THE CHATBOX THE SEED AT NIH
WEBSITE AND THIS IS SMALL
BUSINESS AND SUPPORT OFFICE FOR
ALL OF NIH.
THEIR WEBSITE GOES THROUGH
ELIGIBILITY AND REQUIRED
REGISTRATION AND FAQs AND HELP
POINT YOU TO THE RIGHT FUNDING
TICKETS TO THAT'S A GOOD PLACE
TO RAISE BACKGROUND TO
UNDERSTAND MORE ABOUT THE
PROGRAM AND IF IT'S SOMETHING
YOU WANT TO PURSUE YOU WOULD
ADVISE HAVING A DISCUSSION WITH
A PROGRAM OFFICER AND MYSELF OR
CONTACTS CAN DIRECT YOU TO THE
RIGHT PROGRAM OFFICER WE MAKE
SURE THAT YOU ARE TALK TO GO THE
PERSON THAT YOU WOULD BE WORKING
IF YOU APPLY AND WE ASK FOR THAT
PROJECT DESCRIPTION BUT WE'RE
HAPPY TO TALK ABOUT THE
SPECIFICS OF YOUR APPLICATION
WHAT IT LOOKS LIKE MY DECISION
IS THAT MOST OF THE APPLICATIONS
YOU GET FOR SMALL BUSINESS
GRANTS ARE COMING FROM THEIR
COMPANIES THAT ARE BEING FORMED
BY ACADEMICS LABS IS THAT
CORRECT?
WHAT IS THE DIFFERENCE?
>>
>> YEAH, THEY TAKE A LOT OF
DIFFERENT FORMS BECAUSE THE
ELIGIBILITY IS BROAD.
I WOULD SAY IT'S COMMON THAT WE
ARE FUNDING VERY EARLY STAGES
ACADEMIC SPIN OUTS AND WE HAVE
FILL AND AND COMPANIES THAT WE
ARE FUNDING AS WELL THAT WERE
FORMED BY PARENTS OF CHILDREN
WITH THOSE DISORDERS AND
OTHERWISE HAVE BEEN PERSONALLY
EXPERIENCED DISORDERS THAT
MOTIVATES THEM AND I WOULD SAY
IT'S SOMETHING THAT YOU NIDS IS
LOOKING FOR COMPANIES THAT ARE
DEDICATED TO AREAS SO THAT IS
IMPORTANT FACTOR IN SORT OF THE
COMPOSITION AND I THINK IT'S
VERY FREQUENT THAT WE SEE
ACADEMICS PARTNER WITH NON
PROFITS OR FAMILIES IN SOME WAY
AS WELL AS BUSINESS EXPERTS SO
IT'S NOT LIKE YOU HAVE TO HAVE
ALL THE EXPERTISE WITH YOU AS
ONE INDIVIDUAL AND IT'S ABOUT
RECOGNIZING WHAT STRENGTHS YOU
HAVE AND WHAT YOU ARE BRINGING
TO THE TABLE AND THEN LOOK FOR
THOSE GAPS AND TRY TO FIND AND
WHAT SHE THINKS OF BUILDING A
TEAM THAT'S BEEN SUCCESSFUL.
>> I WAS READING THE CHAT ON THE
SIDELINES.
WHAT DID YOU WANT ME TO ANSWER?
>> I WAS SPEAKING TO THE KINDS
OF TEAMS THAT WE SEE AND HOW
OFTEN THESE ARE VERY EARLY
COMPANIES AND A LOT OF TIMES
ANNAN ACADEMIC IS SPITTING OUT
THE COMPANY AND NOT ALWAYS.
YOU DO NEED TO BE EVERYTHING IN
ONE AS THE INDIVIDUAL STARTING
THE COMPANY AND IT'S ABOUT KIND
OF RECOGNIZING YOUR SKILLS SET
AND THE OTHER SKILLS SETS IN THE
SCIENCE AREA AND BUSINESS AREA
THAT YOU NO HE HAD TO BRING IT
TOGETHER AND I THINK IT'S ABOUT
BUILDING THE RIGHT TEAM IN THAT
IS SUCCESSFUL.
>> SO THAT WAS ONE OF MY
BEGINNING SLIDES.
IT'S A MASSIVE AMOUNT OF WORK
AND YOU JUST HAVE TO IMMERSE
YOURSELF IN IT AND I TRAVEL
QUITE EXTENSIVELY AND I BROUGHT
MY SON WITH ME AND I MET WITH
EVERYBODY AND TOOK A LOT OF
NOTES AND KEPT THE PHONE NUMBERS
AND CALLED THEM BACK AND THAT'S
ONE GOOD THING ABOUT BEING A
PARENT IS THAT PEOPLE LIKE WE
TALKED ABOUT ULTRA-GENICS, I'LL
THROW SOMEONE ELSE OUT THERE
JUDE SMALL SKI, STEVE GRAY,
THESE ARE GREAT ADVOCATES OUT
THERE THAT CAN STEER YOU IN THE
RIGHT DIRECTION AND THAT'S WHAT
YOU NEED.
MY LOCAL HOSPITALS, I'M IN
BROOKLYN, NEW YORK SO I WAS
LUCKY TO BE AROUND NYU AND
EINSTEIN, COLOMBIA, AND GOING
AND TALKING TO THE NEUROLOGISTS
AND THE GENETICIST THAT HAVE NIH
GRANTS AS WELL WHO WORK WITH
ACADEMIA AND OTHER SPONSORS AND
THESE GUYS REALLY GAVE MY
MARCHING ORDERS AND THEY CAN
PROMOTE YOU AND HOOK YOU UP WITH
OTHER PEOPLE AND THAT CAN BRING
YOU FURTHER AND YEAH, YOU JUST
HAVE TO IMMERSE YOURSELF IN IT
AND IT'S HARD AND IT'S A 24/7
JOB AND IT'S YOU KNOW,
ESPECIALLY WHEN YOU ARE TAKING
CARE OF YOUR CHILD.
IT'S NOT RECOMMENDED.
ALSO THE OTHER PATIENT ADVOCATES
AND FAMILIES ARE SUPPORTIVE.
>> WE HAVE A GOOD QUESTION AND
THIS IS IMPORTANT FOR PEOPLE TO
THINK ABOUT BEFORE THEY CREATE A
COMPANY.
AND ANY GUIDANCE ON BALANCING
BETWEEN WANTING THE UPSIDE TO
JUST ROYALTIES ON COMMERCIAL
SUCCESS AND LIMITING FRICTION?
HOW MUCH LEVERAGE DOES IT ARE
AND IT'S A SMART QUESTION
BECAUSE IF YOU ARE HAVING A
COMPANY AND IT MAY BE COMPETING
AGAINST OTHER COMPANIES IN THE
SPACE, HOW DOES IT AFFECT YOUR
ROLE AS A PATIENT ADVOCATE AND
YOUR ABILITY TO SORT OF BE AN
HONEST BROKER WITH ALL OF THE
STAKEHOLDERS IN YOUR DISEASE
SPACE?
>> FOR ANYBODY.
>> SO, LET ME JUST SAY THAT WE
STRUGGLED WITH BALANCING WHAT A
ROYALTY RATES SHOULD BE FOR
CHARITY AND I MEAN, OBVIOUSLY
THE CHARITY'S GOAL IS TO CREATE
A THERAPY FOR THEIR DISEASE AND
ON THE OTHER HAND, THEY HAVE
RAISING MONEY IS HARD AND JILL
TALKED ABOUT IT EASIER AND IT'S
SO YOU HAVE TOUGH TO RAISE MONEY
AND GETTING RETURN IF THERE'S
SUCCESS IS HELPFUL AND SHARING
WITH COMPANIES SO, GENERALLY, I
SORT OF USE A RULE OF THUMB AND
MY RULE IS THUMB IS ANY FUNDING
BELOW LET'S SAY, $8 MILLION I
USE A MULTIPLE OF THE AWARD AND
MULTIPLES RANGE FROM THR THREE O
SIX TIMES AND THEY'RE PAID AT
SUCCESS AT APPROVAL BUT THERE'S
AN EARLY PAYMENT IF THERE'S AN
EXIT TO IT TO A LARGER COMPANY
AND SO WHY IS IT THREE AND NOT
SIX?
DEPENDS IF IT'S EARLY STAGE AND
IN THE RISK IS HIGHER THE
MULTIPLE IS OBVIOUSLY HIGHER SO
IT'S IN THE FIVE-PERSON, FIVE-X
RANGE AND ABOVE THE 8 MILLION
MARK WE COME UP WITH A
PERCENTAGE OF NET SALES.
EVEN IF YOU HAVE A PRODUCT
APPROVED WE PUT SOME GOVERNOR ON
IT AND SOME LIMITATIONS SO WE
SAY IN NO CASE SHALL THE
ROYALTY, SHALL THE FIVE X OR SIX
X IS PAID WITHIN THROW YEARS AND
AFTER A FDA APPROVAL AND USUALLY
WE PUT IN THE NO CASE SHELL THE
PAYMENT EXCEED X.
>> TWO MINUTE WARNING.
>> I WANT TO HEAR YOUR
PERSPECTIVE ON THIS BECAUSE YOU
WERE THE PATIENT ADVOCATE THAT
AND THE HONEST BROKER AND YOU
HAVE TO.
>>
>> YOU TRIGGERED MY MIND ON SO
MANY DIFFERENT THINGS AND
WORKING WITH ACADEMIA CAN BE
BROUGHTALLY CARD AND THEIR TECH
TRANSFER DEPARTMENTS ARE SO HAD
HE BOUGHT BACK AND I GOT ON A
FLIGHT AND BANGED ON THE DOORS
AND SAY NO WAY AND THIS IS
ULTRARARE DISEASE AND THERE'S NO
WAY AND I NEED TO AND YOU HAVE
TO YOU PROBABLY ARE ARE THEIR
ONLY SOURCE AND YOU HAVE OTHER
OPTIONS AND THIS A MATTER OF
GOING OUT FINDING THE RESEARCH
AND IT'S GETTING MARRIED AND YOU
REALLY BEFORE YOU MARRY YOUR
PARTNER AND THEY HOLD THE
STRINGS THAT MAKE LIFE DIFFICULT
FOR YOU AND BROKERING BEING THE
PATIENT ADD CO VISIT AND IT'S
THE THE SPONSOR AS WELL AND IT'S
REALLY NOW CHANGED FOR ME AND I
DON'T DO AK DAMIA RESEARCH SO
MUCH AND WE BRING THEM ON AS
CONSULTANTS SO THEY'RE NOT TIED
INTO THE IF YOU BANK ROLL YOUR
MOUSE MODEL AND DO AND THERE ARE
LOTS OF COMPANIES OUT THERE AND
BUT YOU HAVE TO HAVE CONSULT
APARTMENT WHO IS GOING TO DESIGN
THE ENTIRE STUDY FOR YOU AND BE
WITH YOU ALL THE ENTIRE STEP OF
THE WAY AND I'M SORRY I'M GOING
TO HAVE ALL MY PROFESSOR AND MY
ACADEMICS HELP ME WITH
PITCHFORKS BUT I THINK IF YOU
CAN DON'T THAT AND I WOULD
DEFINITELY DO IT BECAUSE YOU GET
WHAT YOU PAY FOR AND IT'S
SOMEONE GOES ON MATERNITY LEAVE
AND THERE'S SOMEONE TO ACCEPT IN
THE PLACE AND GET MARRIED ALL
THE TIME AND IT'S TOUGH, GUYS.
>> GOOD FOR YOU, JILL.
>> WE HAVE RUN OUT OF TIME AND
EMILY I WANT TO SUGGEST THAT YOU
AT SOME POINT ORGANIZE A WHOLE
DAY WORKSHOP ON SMALL BUSINESS
THERE'S SO MUCH WE NEED TO KNOW.
THANK YOU SO MUCH TO EMILY, KEN,
WE'RE SO GRATEFUL AND JILL FOR
TELLING YOUR STORY AND THANK YOU
FOR JOINING THE PANEL AND SAM,
THINK WE'RE GOING TO A BREAK
NOW, RIGHT.
I'M GOING TO HAND IT BACK OFF TO
YOU.
>> YES, WE ARE.
THANK YOU ALL FOR THAT SESSION
AND THANK YOU FOR MODERATING SO
SHE JUST MENTIONED WE'RE HEADED
INTO A HOUR LONG BREAK UNTIL
2:15.
WELCOME BACK, EVERYONE,
THE AFTERNOON SESSIONS OF OUR
2022NINDS NON-PROFIT QUORUM.
THE SESSIONS THIS AFTERNOON ARE
GOING TO KICKOFF WITH THE ONE ON
YOUR SCREEN.
HOW TO WORK WITH ACADEMIC
RESEARCHERS.
THIS SESSION WILL BE MODERATED
BY Dr. PENNY DACKS FROM THE
ASSOCIATION OF FRONTTOMEPORAL
DEGENERATION WHERE SHE FUNDS
RESEARCH, RECRUIT AND RETAIN TAL
ANT, ENGAGE IN THE COMMUNITY
WITH LIVED EXPERIENCE.
SHE IS PRESIDENT OF THE FTD
DISORDERS REGISTRY LLC WHICH IS
RESPONSIBLE FOR PARTNERSHIPS
SUSTAINABILITY OF THIS
ORGANIZATION AFFILIATE.
PREVIOUSLY, SHE WORKED AT THE
AMERICAN EPILEPSIES SOCIETY
OVERSEEING MISSION RELATED
PROGRAMS IN RESEARCH, MEDICAL
EDUCATION AND CLINICAL
ACTIVITIES AND AS WELL AS AS THE
ALZHEIMER'S DRUG DISCOVERY
PROGRAM WHERE SHE LED COGNITIVE
VITALITY.ORG AND THE AGING
PROGRAM TO SOURCE AND EVALUATE
POTENTIAL THERAPIES.
PENNY TRAINED IN NEUROSCIENCE
WITH A PH.D AND THE ICON SCHOOL
OF MEDICINE IN MOU MOUNT SINAI.
I HOPE PENNY IS ON.
>> YES, I'M HERE.
>> I CAN GO TO YOU AND LET YOU
TAKE IT AWAY.
>> THANK YOU, SAMANTHA.
I'M HONORED TO BE HERE WITH
EVERYBODY TODAY.
SO THINKING ABOUT THE TITLE OF
THIS SESSION LEVERAGING PROGRESS
As COT SCIENTIFIC PIPELINE,
HOW TO WORK WITH ACADEMIC
RESEARCHERS, THAT WE'RE GOING TO
SHOWCASE ARE FEW IS THERE'S NO
ONE ANSWER TO THAT QUESTION.
IT VARIES WHAT IS HAPPENING
WITHIN YOUR SPACE.
THERE IS SOME NON-PROFIT AND
ACADEMIC RESEARCHERS ON WHAT ARE
THE RIGHT STEPS WITH DIFFERENT
ADVISORY BOARDS AND WE FUND
RESEARCH.
HOW DO WE MAKE SURE OUR FUNDING
IS MEETING UNMET NEEDS.
ARE WE ATTRACTING PEOPLE TO THE
SPACE AND ARE WE TRYING TO
BRIDGE FUNDING BECAUSE FUNDING
AVAILABILITY IS BECOME
RELATIVELY WEAK AND ARE WE
SEEING RESEARCH TO GET IT IN OUR
SPACE AND HOW WE WORK CONVENING
THOUGHT LEADERS TO HELP CATALYZE
INNOVATION AND ARE WE CREATING
RESOURCES AND PARTNERING SO THAT
WE CAN FOR EXAMPLE HELP ACADEMIC
RESEARCHERS ACCESS OR UNDERSTAND
THE PATIENT COMMUNITY THAT WE
LIVED WITH AND SO THE RIGHT
ANSWERS TO ALL OF THESE THINGS
DEPENDS ON WHAT IS HAPPENING IN
YOUR FIELD AND WHAT IS THE STAGE
OF RESEARCH AND WHAT IS ON THAT
SCIENTIFIC PIPELINE IN THAT
LONG-TERM JOURNEY WHERE IS IT?
WHERE IS EVERYONE?
AND WHAT IS THE STAGE OF THAT
RESEARCH.
SO WE'RE GOING TO HEAR FROM FOUR
DIFFERENT SCIENTIFIC LEADERS
TODAY ABOUT THE SUCCESS STORIES
THAT THEY'VE SEEN IN THEIR WORK
WITH HOW FAR NON PROFITS CAN
REALLY MAKE A DIFFERENCE AND HOW
THEY CAN HAVE REALLY ROBUST
RELATIONSHIPS WITH ACADEMIC
RESEARCHERS.
WE'RE GOING TO HEAR FROM FROM
KENNETH FISCHBECK AND SABRINA
PAGANONI AND FROM DAVID LIMBRIKK
FROM THE WASHINGTON SCHOOL
UNIVERSITY OF MEDICINE AND
MIGUEL SENA-ESTEVES.
FIRST ON THE LIST IS KEN.
>> CAN YOU HEAR ME OK?
>> SO, I AM A INVESTIGATOR IN
THE INTRAMURAL PROGRAM, THE
DIVISION OF INTRAMURAL RESEARCH
AT NINDS AND THIS IS KIND OF A
UNIQUE SITUATION WE HAVE HERE IN
BETHESDA ON THE NIH CAMPUS.
WE ARE FUNDED TO DO RESEARCH WE
DON'T HAVE TO A REPLY FOR GRANTS
AS OTHER INVESTIGATORS DO IN THE
REST OF THE COUNTRY.
WE ARE FUNDED AND WE GET PAID TO
DO OUR RESEARCH AND WE ARE
REVIEWED EVERY THREE TO FOUR
YEARS BY OUTSIDE REVIEWERS AS
LONG AS WE'RE DOING WELL, WE
CONTINUE TO GET RESEARCH
FUNDING.
WE'RE TRYING TO PUT THAT MONEY
TO GOOD USE AND THE BEST
POSSIBLE USE IN REDUCING THE
BURDEN OF NEWER LO NEURO LOGIC .
I USUALLY SHOW THIS AS
DISCLOSURE AND POTENTIAL
CONFLICTS OF INTEREST BUT FOR
THIS FORUM, I'VE LISTED NOT
CONFLICT OF INTEREST BUT MY TRUE
INTEREST LOWS AND IT'S NOT JUST
ENDURING RESEARCH HERE AT THE
NIH, IN BETHESDA BUT IT'S
ENGAGING IN INTERACTING WITH THE
LARGE NUMBER OF NON-PROFIT
ORGANIZATIONS AND FOR PROFIT
ORGANIZATIONS AND I'VE LONG OVER
THE COURSE OF MY CAREER
INTERACTS WITH IN THE PAST TWO
OR THREE YEARS I'VE INTERACTED
WITH THE ORGANIZATIONS LISTED
HERE AND I'VE BEEN FORTUNATE TO
BE ABLE TO DETHREE SA BAT
TICKALS IN INDUSTRY AND I'M
CONTINUING
THE NON-PROFIT ARE FORECASTED IN
OUR INTEREST OF INTEREST,
NEURODEGENERATIVAL DISEASE AND
THE FRENCH EQUIVALENT OF THE NDA
ASK TREAT-NMD FOUNDATION AND
TARGET ALS, PACKARD CENTER FOR
ALS AND THE KENNEDY'S DISEASE
ASSOCIATION AND RECENTLY AND FOR
SOME YEARS NOW WITH THE SMA
FOUNDATION AND A COUPLE OF
DISEASES THAT FOCUSING ON
HUNTING TON'S DISEASE AND ONE
NON-PROFIT IS THE N-LOREM
FOUNDATION SET UP A COUPLE OF
YEARS AGO AS A PIN SPIN OFF BY E
IONIS FOUNDATION AND IT'S SET UP
LIKE ULTRARARE AND SO ON AND
THIS IS STAN CROOK CAME UP WITH
NANO RARE IT'S A FOUNDATION THAT
IS SET UP TO PROVIDE ALL THE
NUCLEOTIDE THERAPY FOR PATIENTS
WITH DISEASES WHERE THEY'RE THE
ONLY PATIENTS WITH THE DISEASE
IN THE WORLD OR MAYBE UP TO 30
OR SO ARE AFFECTED BY THIS THE
DISEASE SO THEY'RE MUCH LESS
COMMON THAN THE ULTRARARE
DISEASES AND THE FOUNDATION MAKE
THE NUCLEO TIDES AVAILABLE AS
TREATMENT FOR INDIVIDUALIZED HAD
THESE PATIENTS AND PROVIDE THEM
FOR FREE SORRY FAR THEY
WILL DO THAT WITH NEW TREATMENT
FOR THESE PATIENTS THIS YEAR.
AND I MENTIONED THE INTERACTIONS
I'VE HAD WITH THE COMPANIES
LISTED HERE.
SO, NEXT SLIDE.
THE RESEARCH REEF DONE OVER THE
YEARS IS ON THIS PROGRAM WHICH A
SLIDE I'VE USED FOR THIS TIME
NOW AND OVER THE LAST 35 YEARS
NOW, I LARGE NUMBER OF DISEASE
GENES HAVE BEEN IDENTIFIED AND
AT LAST COUNT 7100 DIFFERENT
DISEASES THAT HAVE AN IDENTIFIED
GENETIC CAUSE AND THIS IS BEEN
CONTRIBUTED TO AT THE NIH AND
SEEING PATIENT CHARACTERIZING
THE DISEASE MANIFESTATIONS AND
COLLECTING SAMPLES DURING
SEQUENCING AND ANALYZING THE
VARIANTS AND MAPPING AND
IDENTIFYING THE DISEASE CHAINS
AND CAUSES OF BEGAN ET TICK
DISEASES BUT IT STARTS US ON THE
BOTTOM PART OF THIS CLUB TO
CHARACTERIZE THE GENES AND
FIGURE OUT THE NORMAL FUNCTION
OF THE GENE AND HOW THOSE NORMAN
FUNCTION IS ALTERED IF A LOSS OF
FUNCTION AND TO DEVELOP SUB
CULTURE IN ANIMAL MODELS WE USE
A LOT OF CELLS AND MOUSE MODELS
AND TO USE TO TEST TO SCREEN FOR
NEW TREATMENTS AND THEN TO
ASSESS NEW TREATMENTS FOR
EFFICACY AND SAFETY AND THEN
FINAL LIGHT LAST STEP TO CLOSE
HERE, WHERE THERE HAS BEEN SOME
SUCCESS IT'S BEEN A LOT OF WORK
AND WE CAN POINT TO A DISEASE
THAT'S WE HAVE WORKED ON THAT
HAVE SUCCESSFULLY CLOSED THE GAP
THROUGH CLINICAL TRIALS AND UP
TO HAVE SAFE AND EFFECTIVE
TREATMENT FOR THE DISEASE AND
THE POSTER CHILD FOR OUR FIELD
IS DISEASE WE'VE BEEN INVOLVED
WITH FOR A NUMBER OF YEARS AND
DEFINING THE DISEASE AND HELPING
THE IDENTIFICATION AND CHANGE
AND WORKING WITH OTHERS TO SHOW
BENEFIT IN THE FIRST BENEFITS IN
MOUSE MODELS AND INTERVENTION TO
CORRECT THE UNDERLINE DEFECT OF
DEFICIENCY AND IN MOUSE MODELS
WITH THE DISEASE AND WE HAVE
THROW TREATMENTS AT LEAST THAT T
OF AND BENEFIT WHEN STARTED
EARLIER ENOUGH BEFORE THE
DISEASE ONSET AND THERE'S AN A
NATIONWIDE SCREENING AND TO
IDENTIFY NEWBORN INFANTS WHO
WERE AT RISK.
THE WORK THAT WE DO IS ON THE
NIH CAMPUS HERE IN BETHESDA AND
IT'S DIVIDED BETWEEN BUILDINGS
AND BOTH VERY PORT AT TO BE
USING THESE FACILITIES FOR
LABORATORY RESEARCH AND THE
NEURO SIGN RESEARCH CENTER SHOWN
ON THE NIH CAMPUS LOOKS LIKE A
NIGHT WHEN PEOPLE FORGOT TO TURN
THE LET'S OFF AND IT'S MY OFFICE
IN THE CORNER BUT IT'S WONDERFUL
FACILITY TO DO BABY SEARCH AND
APPLIED RESEARCH RELATED TO
NEUROLOGICAL DEDISEASES AND THE
NIH CLINICAL CENTER OUR BUILDING
10 WHICH IS ONE OF THE LARGEST
IF NOT THE LARGEST RESEARCH
HOSPITALS IN THE WORLD AND IT'S
A WONDERFUL FACILITY TO DO
CLINICAL STUDIES AND
INTERCEPTIONSAL STUDIES PHASE
ONE AND PHASE TWO AND IN
PARTICULAR, AND CLINICAL PROOF
CONCEPT STUDIES WITH THE
DISEASES THAT WE WORK ON AND WE
DON'T HAVE TO APPLY FOR FUNDING
WE HAVE TO GET APPROVAL AND
START THE STUDY AND WE ALSO HAVE
THE OPPORTUNITY TO COLLABORATE
WITH EXTRA MUR AVENUE
INVESTIGATORS AND GETTING THOSE
STUDIES DONE AND TO SHARE IS AND
PROVIDE FUNDING FOR THEM.
I JUST LISTED HERE SOME OF THE
INTRAMURAL CLINICAL
INVESTIGATORS WITH THEIR DISEASE
AREAS AND I THINK ALL OF US CAN
BE VERY USEFUL PARTNERS FOR
NON-PROFIT GROUPS SUCH AS A
REPRESENTATIVE IN THIS FORM AND
COLLABORATORS FOR ACADEMIC
INVESTIGATORS AS WELL THROUGH
SHARED FUNDING AND THIS IS A
SPECIALIST IN PETE ADD TRICK
NEWER LOGIC DISEASES AND MYSELF
FOCUSING ON A RANGE OF NEURO
MUSE KULAR AND PARTICULARLY RARE
HEREDITARY MOTOR NUR ON DISEASES
LIKE KENNEDY DISEASE AND ALS4
AND 8 AND ILS2 AND DAVID
GOLDSTEIN WORKS ON AUTONOMIC
DISORDERS AND THERE'S A STROKE
BRANCH AND MARK HAS A LONG AND
VERY PRODUCTIVE CAREER IN
MOVEMENT DISORDERS RESEARCH AND
NEW RECRUIT WORKS ON PARK
INSINCE DISEASE AND JUVENILE
VARIANTS AND THE CLINICAL
DIRECTOR SO HE IS IN CHARGE OF
ALL THIS GROUP AND HAS A
PARTICULAR INTEREST IN NEURON
INFECTIOUS DISEASES SUCH AS
COVID AND HIV.
DAVE REICH HAS BEEN IN THE
BRANCH WITH A FOCUS ON MULTIPLE
SCLEROSIS AND SONIA SCHOLZ WORKS
ON LEWY BODY DEMENTIA BETWEEN
ALZHEIMER'S AND PARKINSON'S AND
MICHAEL WARD AND A NEW
UP-AND-COMING INVESTIGATOR IN
OUR GROUP WHO WORKS ON ALS AND
IT'S INTIMATELY CONNECTED WITH
THE INTRAMURAL PROGRAM CALLED
CARVE A CENTER FOR ALZHEIMER'S
AND RELATED DISORDERS AND AND HE
RUNS A SUB CULTURE PROGRAM
PROJECT THROUGH CARVE AND HE IS
INVOLVED WITH THAT WHICH IS VERY
WELL FUNDED THROUGH THE NATIONAL
INSTITUTE OF AGING AND KAREEM A
STAR NEUROSURGEON AND WHO IS
GROUP WORKS ON BRAIN TUMORS AND
HE IS ALSO INVOLVED IN SURGICAL
TREATMENT FOR EPILEPSIES SO
AWFUL THESE PEOPLE ARE PEOPLE
YOU CAN GET IN TOUCH WITH AND I
ENCOURAGE YOU TO FORM A WORKING
RELATIONSHIP WITH THIS
INVESTIGATORS TO MOVE THE
FORWARD.
THERE ARE A NUMBER OF OTHERS WHO
ARE STAFF CLINICIANS AND
ASSISTANT CLINICAL INVESTIGATORS
AND IT'S A USEFUL RESOURCE TO
TAP INTO.
SO HOW DO YOU FIND IN GENERAL,
HOW DO YOU FIND CLINICAL
ININTEREST MURAL THAT YOU CAN
WORK AND LOOKING AROUND AT
DIFFERENT WEB SITES I THOUGHT
MOST USEFUL IS A WEBSITE ON
PATIENT RESEARCH AT NINDS AND I
GIVE THE LINK HERE BUT IF YOU
JUST GOOGLE ON OUR SEARCH ON
PATIENT RESEARCH YOU WILL GET
THE LIST I SHOWED YOU AND THE
CONTACT INFORMATION HOW TO GET
IN TOUCH WITH PEOPLE O'ER YOU
CAN GO TO THE OFFICE OF THE
CLINICAL DIRECTOR THAT IS Dr.
AL BYE AND HIS OFFICE CAN SEND
YOU FURTHER AND JUST SEARCH ON
CLINICAL DIRECTOR OR SPEAKING OF
SEARCHES A TRICK I PICKED UP A
FEW YEARS AGO IS YOU CAN JUST
PUT IN THE NAME OF YOUR DISEASE
AND A SEARCH AND THE ZIP CODE
20892 WHICH IS A UNIQUE ZIP CODE
FOR THE NIH CAMPUS AND IT'S
GOING TO TAKE YOU TO
PUBLICATIONS AND HOME PAGES FOR
RESEARCHERS AT THE NIH AND
WHATEVER AREA OF YOUR INTEREST
AND THEN FINALLY, ALL ELSE
FAILS, CONTACT ME AND YOU CAN
SEARCH ON MY NAME.
AND YOU WILL GET MY E-MAIL THE
NIH IS A PIECE OF THE JIGSAW
PUZZLE BUT ANY OTHERS THAT ARE
SHOWN HERE.
PARTICULARLY THE DISEASE
FOUNDATION IS A REPRESENTATIVE
THEY PUT THE WIND IN OUR SALES
AND YOU ARE CUSTOMERS AND OUR
BOSSES BECAUSE OUR FUNDING COMES
THROUGH CONGRESS WITH HELP FROM
PATIENT ADVOCACY GROUPS AND WE
DEPEND ON YOU THROUGH CONGRESS
AND CONGRESSIONAL APPROPRIATIONS
AND YOU ARE ALSO DEVOTED TO
SERVING AND MEETING YOUR NEEDS,
EFFECTING THOSE EFFECTING
YOURSELVES OR YOUR CHILDREN AND
YOUR FAMILY MEMBERS AND WE ALSO
HAVE TO ENGAGE IN THIS AND I SEE
IT'S LIKE A RELAY RAISE AND I
THINK THIS IS TRUE OF ACADEMIC
RESEARCHERS IN GENERAL AND
SHOWING EVIDENCE AND THEY'RE
GOING TO WORK AND THAT STAGE WE
HAVE TO PASS THE BATON ON TO
INDUSTRY TO CARRY IT THROUGH TO
A DRUG YOU CAN GO TO THE
DRUGSTORE AND GET AND GET THE
ADMINISTERED IN THE HOSPITAL 20
GET FDA APPROVAL TO PAY FOR IT
BUT INTER MIGHT WITH INDUSTRY
AND IT'S JUST A POINT ON THIS
SLIDE BUT IMPORTANT RELATIONSHIP
WITH OUTSIDE ACADEMIC MEDICAL
CENTER AND NIH IS THE MAIN
SOURCE OF FUNDING AND MANY OF
THESE RESEARCHERS AND WE'RE
HAPPY TO COLLABORATE WITH
OPPORTUNITIES FOR CO FUNDER AND
I HOPE I WAS ON TIME.
I DIDN'T RUN OVER TOO MUCH.
>> THANK YOU.
WE'RE GOING TO HOLD ON QUESTIONS
UNTIL ALL FOUR SPEAKERS HAVE A
CHANCE TO CHAT AND WHILE YOU ARE
LISTENING TO ANY OF THESE
SPEAKERS ININCLUDEDDING, PLEASE
GO AHEAD AND PUT QUESTIONS IN
THE CHATBOX AND WE'LL MAKE
THOUSAND THOSE GET ADDRESSED AS
MUCH AS TIME ALLOWS AT THE END
OF THE SESSION AND OUR NEXT
SPEAK SER Dr. DAVID DIM BRICK
AND THE CHIEF OF PEDIATRIC NEURO
SURGERY AND EXECUTIVE VICE-CHAIR
AT WASHINGTON UNIVERSITY.
HE ALSO SERVES AS THE
NEUROSURGEON AND CHIEF AT ST.
LOUIS CHILDREN'S FONT AND GO
RIGHT A HEAD, PLEASE, LET ME FOE
IF YOU HAVE ANY TECHNICAL ISSUES
WITH MY DECISION AND TODAY MY
TOPIC IS GOING TO BE THE ROLE
AND INVOLVEMENT OF NON-PROFIT
ORGANIZATIONS IN PATIENT
ORIENTED RESEARCH AND SO I'M
HAPPY TO TALK TO YOU ABOUT THIS
TODAY.
JUST TRYING TO ADD THESE SLIDES
AND I DON'T KNOW IF I DID THAT
BUT I HAVE SOME DISCLOSURES THAT
ARE DEVELOP RANT TODAY AND THIS
SLIDE JUST KIND OF GOES THROUGH
FUNDING SOURCE AND SO FOURTH BUT
THE RELEVANT DISCLOSURES WHICH
I'LL TRY TO GET BACK TO AND KEEP
MY STREAM OF THOUGHT INCLUDED
AND WHEN EXECUTIVE COMMITTEE AND
AND FOUNDATION AND I JUST WANT
TO SHOUT OUT TO THEM BECAUSE
THEY'RE ON THE LINE TODAY AND
MARY, KAITLYN AND DORTHY THANK
YOU FOR ATTENDING ONE OF THE
MOST CONTROVERSIAL QUESTIONS IN
PEDIATRIC NEURO SURGERY AROUND
THE THEY ARE DEPLOYED FOR THAT
AND ONE IS IS I BONE AND BIGMENT
AND THE OTHER ONE IS A STEP
FURTHER WHERE WE OPEN UP WE
APPLIED THROUGH THE INSTITUTE
AND IN THE FUNDING WAS TO
PERFORM A RANDOMIZED CONTROL
TRIAL COMPARE THESE TWO
DIFFERENT OPERATIONS WHICH HAVE
A DIFFERENT RISK PROFILE AND A
DIFFERENT EFFICACY PROFILE AND
I'M GOING TO SAY I'M HAVING
TROUBLE MOVING THEM FORWARD AND
THANK YOU.
AND THIS TRIAL WAS SUPPORTED
THROUGH ALL THE MAJOR AT TRICK
ORGANIZATION AND IT'S A CLUSTER
RANDOMIZED AND A HIGH-QUALITY
LAST YOU CAN SEE THE ENROLLMENT
IN BLUE AND THERE ARE A LOT OF
FACTORS INVOLVED IN THAT AND ONE
KEY THING AND THIS IS WHAT I
WANT TO HIGHLIGHT HERE IS THAT
THEY ARE DEVELOP RANT FROM THE
PATIENT COMMUNITY AND DO YOU
MIND ADVANCE INTERESTING IF YOU
DON'T MIND I'M GOING 20 TRY THIS
METHOD.
AND THANK YOU AND SO WE WENT
THROUGH A NUMBER OF DIFFERENT
ENGAGEMENT ACTIVITIES TO TRY TO
FOSTER SUPPORT IN THE TRIAL AND
CAN YOU ADVANCE THE SLIDES MORE
AND THIS IS ONE EXAMPLE OF THIS
WAS PUT ON BOBBIE JONES AND OUR
SAINT AND FLOOR THIS AND WE
REALLY WORKED ON THE LEGAL
SUPPORT FOR FAMILIES AND DEALING
WITH A CHILDHOOD IS DIS ABILITY
AND THE EDUCATIONAL SUPPORT AND
YOU CAN GO FORWARD ONE NOW AND
ONE OF THE THINGS THAT TURNED
OUT TO BE ABSOLUTELY KEY IN OUR
ENROLLMENT AND OUR RELATIONSHIP
WITH BOBBIE JONES AND WHAT
HAPPENED EARLY IN THE TRIAL ON
THUR SOCIAL MEDIA ACCOUNTS THEY
STARTED TO SEE SOME NEGATIVE OR
NEAR NEGATIVE COMMENTS ABOUT
PARTICIPATING IN THE CLINICAL
TRIAL AND WHAT THEY DID WAS THEY
REALLY HELPED US TO DISPEL
NEGATIVE FEELINGS REGARDING
CLINICAL RESEARCH AND THROUGH
ON-LINE CAMPAIGNS HERE IS AN
EXAMPLE AND YOU CAN ADVANCE AND
THEY HELPED US TO CREATE PATIENT
BROCHURES TO REASSURE PATIENTS
THAT THERE'S POINTS BETWEEN
THESE TWO DIFFERENT PROCEDURES
AND WE REALLY NEEDED TO SUPPORT
THE RESEARCH THERE AND THERE'S
AN EXAMPLE OF PATIENT PRO SURE
IF YOU CAN ADVANCE ONE MORE
SLIDE, WITHOUT THEM WE WON HAVE
MET OUR IT WOULD HAVE BEEN
UNDER-POWERED SO YOU CAN MOVE
FORWARD, PLEASE.
THE SECOND EXAMPLE IS ONE IN
HYDROCEPHALUS.
YOU KNOW THE TWO KEY TREATMENTS
FOR HYDROCEPHALUS IN CHILDREN IS
YOU MAY BE AWARE IS SHUNTING, AS
YOU CAN SEE ON THE LEFT WHICH IS
IS A LITTLE TUBE OR A NEW
PROCEDURE WHICH WE'LL USE THE A
ABBREVIATION OF ETV-CPC FOR AND
YOU CAN ADVANCE ONE MORE SLIDE
THERE AND PATIENT POPULATIONS
FOR THIS AND WHAT IS HAPPENING
IS DESPITE A LACK OF EVIDENCE
THAT ONE IS BETTER THAN THE
OTHER THERE'S A GLOBAL RISE OF
THE USE OF THIS PROCEDURE AND
YET, THE VEN TRICK ALS, THE
FLUID CAVITIES REMAIN LARGE WITH
ATV SO WE'RE NOT SURE WE WORKED
WITH THE HYDROCEPHALUS AND
BEFORE WE DESIGNED THE TRIAL
WHAT'S MOST IMPORTANT TO
FAMILIES WITH HYDROCEPHALUS AND
THE MOST IMPORTANT FACTOR WAS
COGNITIVE FUNCTION.
SO THERE WAS A STRONG DESIRE FOR
FAMILIES TO HAVE THEIR CHILDREN
BE FREE OF SHUNTS BUT THEY WERE
EVEN WILLING TO UNDERGO MORE
THAN ONE SURGICAL PROCEDURE TO
BE SHUNT FREE AND IF IT DIDN'T
EFFECT COGNITIVE FUNCTION AND
IT'S A KEY FACTOR IN THE
DECISION-MAKING HERE SO WE
DESIGNED AN RCT TO HELP US TEST
THIS AND THIS LIGHT ON THE RIGHT
I JUST WANT TO SAY THAT ONE
THING THAT HA HAS DONE REALLY
WELL IS THEY'VE CREATED A SERIES
OF INTER LACED EFFORT.
ONE IS CRITICAL RESEARCH
NETWORKS AND A DISCOVERY SCIENCE
NETWORK AND THERE'S THE PATIENT
POWERED REGISTRY WHICH IS
HYDROCEPHALUS ASSOCIATION
PATIENT-POWERED I'M NOT SURE OF
THE INE ARE SO YOU CAN GO
FORWARD, PLEASE.
SO WE DO HAVE THIS RCT NOW THAT
REALLY TAKES THE INFORMATION
THAT WE GOT FROM THE
HYDROCEPHALUS SU SURVEY TO DESIN
THIS RCT WHICH IS U01 FUNDED
THROUGH THE NINDS AND THE
PRIMARY OUTCOMES WHICH WAS
GENERATED WITH THAT INFORMATION
FROM HA IS THE PRIMARY OUTCOME
IS COGNITIVE OUTCOME AT 12
MONTHS SO FOR NEUROSURGERY WE
MIGHT SAY THE PRIMARY OUTCOME
WOULD BE SHUNT REVISION OR
INFECTION OR SO FOURTH BUT SO I
DO WANT TO SAY THANK YOU FOR ALL
THE SUPPORT FOR THE
HYDROCEPHALUS ASSOCIATION AND I
CAN SEE SOME OF THE MEMBERS ARE
HERE O AN AS WELL I'M GRATEFUL
TO BE INVOLVED WITH THIS
INFORMATION WITH THIS
ORGANIZATION AND IN FACT I'M ON
THE BOARD OF DIRECTORS AS I SAID
BEFORE AND WE JUST HAD OUR
NATIONAL CONFERENCE THE LAST
WEEKEND WHICH IS A SUCCESS SO
CONGRATULATIONS SO YOU CAN MOVE
FORWARD, PLEASE.
I WANT TO SAY IN 2022, YOU JUST
SAW A SIMILAR IMAGE IN KEN'S
PRESENTATION BUT IN 2022, TO
MAKE THE KINDS OF ADVANCES AND
SCIENCE AND CLINICAL MEDICINE
THAT WE NEED TO, IT REALLY TAKES
A TEAM AND SO THAT TEAM IS
PEOPLE WITH MULTIPLE DIFFERENT
SPECIALITIES WITH DIFFERENT
EXPERIENCES AND DIVERSE SKILLS
AND ALL FOCUSED ON A COMMON GOAL
AND AND THEY ARE KEY PLAYNESS
OUT WE DO TEAM SIGNS AND WE HAVE
CLINICAL TRIALISTS AND CLINICAL
EXPERTS AND THEN A KEY PIECE OF
THE PUZZLE IS INPUT FROM
PATIENTS AND ADVOCACY GROUPS AND
WITH THAT I'LL STOP AND I'LL
WAIT FOR THE QUESTIONS TO COME
UP DURING QUESTION PERIOD LATER
AND THANK YOU SUCH FOR YOUR
ATTENTION.
>> IT'S INTERESTING TO SEE THE
EXACT SAME META FOR FOR YOURSELF
ACROSS DIFFERENT TYPES OF
STAKEHOLDERS AND I THINK IT'S
GOING TO BE SOMETHING WE SHOULD
CIRCLE BACK TO ABOUT THE WAYS OF
NON PROFITS CAN HELP WITH THOSE
DIFFERENT PIECES FITTING
TOGETHER.
WE HAVE THE CO DIRECTOR THE
NEUROLOGICAL INSTITUTE AT THE
MASSACHUSETTS GENERAL HOSPITAL
AND HARVARD MEDICAL SCHOOL HER
RESEARCH IS ON CLINICAL TRIALS
AND THERAPY DEVELOPMENT.
TAKE IT AWAY.
>> THANK YOU SO MUCH AND IT'S
SUCH A PLEASURE TO BE HERE WITH
ALL OF YOU AND AS YOU SAID, A
CLINICAL TRIAL IS FOCUSING ON
ALS AND TODAY I WAS ASKED TO
REFLECT AN THE IMPORTANCE OF OUR
RELATIONSHIPS WE FOUNDATIONS SO
I WANT TO FOCUS ON THAT AND
START FROM THE BEGINNING OF MY
CAREER WHERE IT'S BUILDING ON
THE SAME META FOR WE'RE HERE
BECAUSE OF OUR AND FOUNDATIONS
WE ARE ALL WORKING FOR THE SAME
GOAL SO THE WORK I'VE BEEN DOING
DID REALLY COLLABORATIVE BETWEEN
THE FDA SCIENTISTS AND
FOUNDATIONS HAVE BEEN SUPPORTING
MANY OF OUR ACTIVITIES AND I
WANT TO TALK ABOUT A DIFFERENT
CONTEXT AND DIFFERENT WAYS THAT
FOUNDATIONS CAN SUPPORT ACADEMIC
PHYSICIAN INVESTIGATORS LIKE
MYSELF.
SO GOING BACK TO THE FINING, I'M
DOING CLINICAL TRIALS AS MY MAIN
OCCUPATION GOES BACK TO A
CLINICAL SCIENTIST DEVELOPMENT
AWARD I GOT IN 2017 AND I'M
THERE IN THE MIDDLE YOU MAY
NOTICE DIFFERENT HAIR COLOR BUT
I CHANGE THAT FREQUENTLY SO I'M
IN THE MIDDLE WITH MANY OTHERS
ARE STILL GREAT FRIENDS AND
COLLABORATORS AND WE REALLY HAVE
A CLASS OF FUNDED INVESTIGATORS
AND STARTING TOGETHER IN 2017
AND EVERYONE HAS BEEN
SPECIALIZING IN DIFFERENT AND
NEUROLOGICAL AREAS AND THE
DEVELOPMENT AWARD I GOT WAS
SUPPORTED BY THE ALS ASSOCIATION
IN COLLABORATION WITH THE
AMERICAN ACADEMY OF NEUROLOGY
AND THE AMERICAN BRAIN
FOUNDATION AND IT'S REALLY
WONDERFUL THAT DIFFERENT
ASSOCIATIONS COME TOGETHER TO
FUND THIS SPECIFIC RESEARCH AND
IN MY CASE SPECIFICALLY, I
FINISHED MY TRAINING INCLUSIVE
RESIDENTIAL BOARD CERTIFICATES,
ET CETERA, WHEN SOMEONE FINISHES
THE TRAINING AS A PHYSICIAN
SCIENTIST IT'S A TIME IN ONES'
CAREER WHERE YOU HAVE TO GET
PROTECTIVE TIME TO REALLY
DEVELOP EXPERTISE AND A
PHARMACEUTICAL COMPANY IN THE
BOSTON AREA BUT THE FUNDING, THE
INITIAL FUNDING FOR SOME OF THE
INITIAL EXPERIMENT THE SUCH AS
TOXICOLOGY AND SOME OF THE
INITIAL INFRASTRUCTURE GRANTS
FOR THE TRIAL AND WAS FUNDED
ACTUALLY BY A FOUNDATION AND
THIS TRIAL WOULD HAVE NEVER
HAPPENED WITHOUT THE HELP AND
NOW AFTER THAT, AFTER THE
PROVIDED THE FUNDING, THE
COMPANY WENT ON TO SECURE
ADDITIONAL FUNDING OF VENTURE
CAPITOL, ET CETERA, THEY DID A
GREAT JOB EXPANDING THE TRIAL
AND HERE WE ARE AT FIVE YEARS
LATER WHERE THE DRUG THAT WAS
TESTED IN THAT TRIAL WAS
ACTUALLY APPROVED BY HEALTH
CANADA THAT HAPPENED A FEW WEEKS
AGO AND WE EXPECT THE FDA
DECISION BY SEPTEMBER AND
THERE'S AN ADDITIONAL CONFIRM
TORI TRIAL THAT'S ON GOING IN
EUROPE.
IT HELPED GET THIS PROGRAM OFF
THE GROUND AND NOW WE'VE BEEN
APPROVED DRUG IN CANADA AND
HOPEFULLY APPROVED DRUGS IT'S
THE FIRST PLATFORM TRIAL FOR ALS
SO IT'S A LARGE-SCALE TRIAL
WHERE YOU TUFT DRUGS STATEMENT
SO RIGHT NOW WE'RE TESTING FIVE
DRUGS FOR ALS USING THE SAME
INFRASTRUCTURE AND WE'RE ADDING
MORE DRUGS TO THE SAME PLATFORM
AND THE TRIAL IS NOT DIRECTOR
THE CENTER FOR ALS AND GENERAL
WHERE I'M SPEAKING FROM AND THE
REASON THERE ARE LOGOS ON THE
SLIDE IS BECAUSE THIS IS TEAM
SCIENCE AND WE ARE DOING THIS IN
COLLABORATION WITH MULTIPLE
STAKEHOLDERS AND SOME OF THEM
ARE ACADEMIC DOLL COLLABORATORT
AS YOU SEE THERE'S A NUMBER OF
FOUNDATIONS THAT ARE SUPPORTING
PIECES OF THE TRIAL AND THIS IS
CRITICAL FOR THE CONDUCT OF THE
TRIAL ITSELF AND REALLY BEING
ABLE TO TEST SO MANY DRUGS AT
THE SAME TIME IN THE SAME
DISEASE AREA THEY MAY NOT BE
THEY ARE FOR ANT FOR PATIENTS
AND ACADEMIC RESEARCHERS SO FOR
EXAMPLE, FUNDED BY FOUNDATIONS,
SPECIFIC FOUNDATIONS WE ARE
FOCUSING ON INCLUSION AND NOW WE
HAVE AND HAVE PEOPLE WHO CAN
ANSWER QUESTIONS FROM PATIENTS
LIFE AT ALL TIMES AND BECAUSE OF
THEIR HELP, WE HAVE CONNECTED
WITH THOUSANDS OF PATIENTS OF
ALS AND THIS IS REALLY
RESPONSIBLE IN THE LARGE PART IN
2020 WE'VE HAD GREAT ENROLLMENT
RATES AND WITH THE PATIENT
COMMUNITY AND SPECIFIC ASPECTS
OF THE TRIAL THAT ARE FUNDED BY
SPECIFIC ORGANIZATIONS AND I'M
NOT GOING INTO THE DETAILS BUT
ACCESS IS AND INCLUSION IS
BIOMARKERS AS OTHER SO I THINK
FOR THE WORK WE DO COLLABORATING
WITH FOUNDATIONS AND PATIENTS
CENTER GROUPS, IT'S CRITICAL AND
FOR ACADEMIC INVESTIGATORS LIKE
MYSELF IT'S CRITICAL WITH
SPECIAL EXPERTISE IN ONE DISEASE
AREA.
THAT WAS ALL I HAD TO SHARE OUR
LAST SPEAKER IS Dr. MIGUEL HE
IS A ASSOCIATE PROFESSOR IN THE
GENE THERAPY CENTER AT U.S. CHAN
MEDICINE SCAL SCHOOL AND AND AS
WELL AS GENE THEY ARE FEE FOR
DISEASES AND I WILL LEAVING TO
HIM TO EXPLAIN MORE.
GOOD AFTERNOON, EVERYONE.
I HAVE NO SLIDES TO SHOW THIS
AFTERNOON SO I DON'T RUN THE
RISK OF REPEATING SOME OF THE
ASPECTS THAT MY COLLEAGUES HAVE
ALREADY TALKED ABOUT IT'S
COMPLICATED AND THERE'S NO
QUESTION THAT IN SCIENCE AS WE
BECOME MORE AND MORE COMPLICATED
WITH BRINGING TO BEAR LOTS OF
DIFFERENT TECHNOLOGIES SCIENCE
IS PART OF A LARGE TEAM TO BRING
THE TECHNOLOGIES WE HAVE AT HAND
TO BEAR IN PARTICULAR QUESTIONS
AND SO, INSTEAD OF GOING ON A
TANGENT THAT I DID WHEN I SPOKE
TO PENNY BEFORE, I AM ACTUALLY
GOING TO TELL YOU MORE ABOUT MY
EXPERIENCE ON WORKING WITH ONE
FOUNDATION TO DEVELOP A GENE
THERAPY FOR A DISEASE THAT IS A
WELL-KNOWN INHERITED DISEASE
DESCRIBED MANY YEARS AGO.
WHEN I STARTED ON THIS PROJECT,
ACTUALLY IT STARTED FROM THE
VERY BEGINNING BY BRINGING
TOGETHER QUITE A FEW SCIENTISTS.
IT HAD DIFFERENT PIECES OF THE
PUZZLE, IF YOU WILL, THAT WE'RE
WORKING ON MOUSE MODELS, LARGE
ANIMAL MODELS FROM THE BEGINNING
IT WAS GOAL THE BRINGING
EVERYONE TOGETHER AND THEY HAD
PLAY A CENTRAL ROLE BECAUSE I
WAS ABLE TO BRING TOGETHER
INDIVIDUALS AND PROVIDE SEED
FUNDING TO GET THE PROJECT
STARTED.
THERE WAS NOTHING IN THE OR EYES
ON THAT COULD CHANGE THE AND SO
WE STARTED OUT RESEARCH WITH
SOME OF THE FUNDING FROM NTSAB
AND WAS ME AND A BUNCH OF
DIFFERENT COLLEAGUES ACTUAL WILL
HE FROM ABROAD AS WELL AND AT
BEGINNING EVENTUALLY WE WERE
ABLE TO WRITE A GRANT TO NIH AND
TO SUPPORT MOVING OUR FINDING
FROM THE BEACH TOWARDS THE
CLINIC AND DOING TOXICOLOGY
STUDIES, ET CETERA.
WHAT WE FOUND OVER THE COURSE OF
A COUPLE OF YEARS, IN OUR WORK,
IS THAT WHILE SOMETHING THAT
FAPHARMACOLOGISTS KNEW, IF YOU
GIVE CELLS IN THE BRAIN, ASK
THEM TO MAKE TOO MUCH OF A GOOD
THING, IT'S A BAD THING AND WE
KNOW THAT FROM TILE INOLE OR
ADVIL BUT IF YOU TAKE TOO MUCH
IT'S A MAJOR PROBLEM.
WE FOUND THAT OUT AT THE GENETIC
LEVEL AND THAT IS WHERE THE
PROGRAM COULD HAVE DIED.
THAT'S WHERE IT COULD HAVE
STOPPED AND THEY RAISED A LOT OF
MONEY AND WAS ABLE TO PROVIDE
FUNDING NECESSARY TO CONTINUE
THE PROJECT TO THE POINT WE SAW
THOSE TOXICOLOGY ISSUES WE HAD
ENCOUNTERED AND FIND AND DEVELOP
NEW VECTORS THAT EXPRESS OR MAKE
THE NORMAL PROTEINS AT LOWER
LEVELS BUT DON'T KILL THE CELLS
WHICH IS THE PRIMARY GOAL THAT
DEVELOPED A TREATMENT THAT DOES
NEED TO PROCESS KILL CELLS IN
THE BRAIN BECAUSE THEY DON'T
COME BACK WHEN THEY'RE DEAD AT
LEAST FOR THE MAJORITY OF THE
CASES.
WITH THAT IN MIND, WE SPEND
YEARS AND THAT WERE CONSULTED
FOR THESE FOUNDATIONS FROM
INDUSTRY TELLING US THAT WE
ABSOLUTELY NO ABILITY TO TAKE
THIS TO A CLINICAL TRIAL AND IT
MAY HAVE BEEN TRUE BUT MONEY WAS
NOT AVAILABLE AND WE'RE NOT
INTERESTED IN GREEN THERAPY IT'S
THIS MAYBE IT WORKS BUT WE NEED
LARGER INDICATIONS BECAUSE IT
FALLS INTO DISEASE AND IT'S HARD
TO MAKE A CASE FOR ULTRARARE
DISEASES AND WE'RE STUCK IN THAT
CYCLE OF YOU CAME TO IT BUTNTTEN
DOING IT SO, A FOUNDATION CAME
ON BOARD, THE BLUE JEANS
FOUNDATION THAT ALLOWED US TO
TAKE THE FINAL STEPS TO GET TO
CLINICAL TRIAL AND WE WERE ABLE
TO GENERATE CLINICAL TRADE
MATERIAL WHICH ARE EXPENSIVE AND
WITH ADVENTURES THEY'RE SINGLE
COULD LAST FOR A LONG TIME AND
WE SEE WHAT HAPPENS IN MODELS
AND WE DID ALL THE STUDIES FOR
THE FDA TO APPROVE THE PILE AND
WE HAVE TWO PATIENTS AND WE SAW
BENEFITS AND WE STARTED WITH
VERY LOW AND SINGLE TO THE
TREATMENT THAT CAN'T BE TAKEN
AWAY WHEN YOU PUT IN IT STAYS IN
THE CELLS AND EVENTUALLY THE
TECHNOLOGIES WERE LICENSED AND
ALL OUR DATA THAT HAD SUPPORTED
THAT INITIAL CLINICAL TRIAL WAS
A PHASE TO STUDY THAT IS STILL
ON GOING TODAY SO ONE OF THE
THINGS WE'VE DONE IS
PARTICIPATED IN THE ANNUAL
MEETING OF THE FONDATION IT'S
CALLED THE FAMILY MEETING TO
ACTUALLY MEET THE PATIENTS AND
NOT ONLY MEET THE PATIENTS
ACTUALLY EDUCATE THEM AS WELL IN
WHAT WE'RE TRYING TO DO WITH THE
TECHNOLOGY.
OVER THE YEARS, YOU WOULD START
TO GET QUESTIONS THAT WERE
EXTREMELY SPECIFIC TO THE
TECHNOLOGY SO THERE'S VERY, VERY
CLEAR THAT INDIVIDUALS THAT
PARTICIPATED IN THOSE MEETINGS
ON A YEARLY BASIS STARTED TO
REALLY UNDERSTAND THE BASICS OF
THE TECHNOLOGY AND THAT IS ALSO
IMPORTANT FROM THE POINT OF VIEW
OF THE PATIENT COMMUNITY TO
REALLY HAVE A GOOD SENSE OF WHAT
IS IT THAT IS BEING DONE?
ON THE SURFACE IF YOU LOOK AT T
THE IDEA OF PUTTING GENES BACK
INTO YOUR BODY CAN BE SCARY.
NOW A LAYS A LITTLE BIT LESS BUT
NONE THE LESS, IT'S STILL A
LITTLE BIT OF A DAUNTING IDEA.
IT'S NECESSARY THE COMMUNITY
GETS EDUCATED, LEARNS THROUGH
THE RESEARCH SOURCE THAT THERE'S
A DEGREE OF TRUST THAT IS PRETTY
IMPORTANT TO DEVELOP OVER TIME
AND WE CONTINUE TO GO TO THE
FAMILY MEETINGS AND ARE
COMPLETELY TRANSPARENT WITH THE
FAMILIES AND THE FOUNDATIONS
WHERE WE ARE TODAY AND WHAT ARE
THE PROBLEMS THAT WE HAVE AND
FORTUNATELY NOT MANY NOWADAYS
AND WE CONTINUE TO WORK VERY,
VERY CLOSELY WITH THIS
FOUNDATION SO BASED ON THIS
EXPERIENCES, IT'S A SECOND
PROGRAM FOR ANOTHER STORAGE
DISEASE THAT ACTUALLY WE
COLLABORATE WITH A COLLEAGUES
THAT WORKS IN THE BUILDING 10,
THE CLINICAL RESEARCH CENTER AT
NIH SO THIS IS A NICE STARTED
WITH AND SO BASED ON THIS
EXPERIENCE, WE TOOK THAT PROGRAM
AND DEVELOPED IT ALL ON OUR OWN
SO WE WORD THAT YES ACADEMICS
CAN AND DEVELOP AND LEAD
PROGRAMS TOWARDS CLINICAL TRIALS
AND IT REQUIRES A LOT OF
PERSONAL AND THERE'S NO QUESTION
ABOUT THAT AND YOU HAVE IF HE
TAKE YOUR OWN HANDS AND
DO-IT-YOURSELF AND YOUWORK WITH
THE THINGS ARE DONE PROPERLY AND
OF COURSE THEY HAVE TO FIT THE
CRITERIA AND THE REQUIREMENTS
THAT THE FDA HAS BASED ON THIS
EXPERIENCE TO START THE
TRANSLATIONAL INSTITUTE FOR
THERAPEUTICS UMAS SCHOOL TO
TRANSLATE THAT EXPERIENCE TO
TRANSLATE FROM THE BENCH TO THE
CLINIC IN ANNIE MISH MANNER AND
WHAT WE UNDER UP WORKING ON ARE
RARE DISEASES AND WE'RE
CONTACTING BY PATIENTS AND THE
PARENTS AND WE STARTED TO FORM
PARTNERSHIPS WITH THE FAMILIES
AND WE ALWAYS ADVISE THEM TO
FORM A FOUNDATION AND THERE CAN
BE A FORMAL THEY STRUGGLED TO
RAISE MONEY BECAUSE WE'RE
TALKING ABOUT AGAIN LIKE DISEASE
WHERE THERE MAY BE 10 PATIENTS
KNOWN WORLD WILD AND WE CAN MAN
MAKING A COMMERCIAL CASE IS A
BIT DAUNTING AND I DON'T MAKE
THAT COMMERCIAL CASE SO WE'RE
WORKING VERY CLOSELY WITH THE
FOUNDATION THAT WAS STARTED BY A
FAMILY AND THIS IS ACTUALLY
EXAMPLES OF THAT WE HAVE NOW ON
GOING AND TO RAIL' TAKE IT TO
THE SMALL CLINICAL TRIALS AND
HOPEFULLY SHOW EFFICACY AND
MAYBE THERE'S SOME THAT WANTS TO
TAKE IT OVER AND O NEVER IT OVER
AND THESE THERAPIES HAVE MADE IT
AVAILABLE AND THAT SUFFER FROM
RARE CONDITIONS THAT MAY IN FACT
HAVE THE CATEGORY OF THE RARE
DISEASES AND ULTRARARE DOESN'T A
PLOY TO THIS ONE SO OUR
EXPERIENCE HAVE BEEN BROAD AND I
WORK WITH FOUNDATIONS THAT HAVE
SUFFICIENT FUNDS THAT REALLY CAN
THROW MANY ERRORS AT LODGE OF
TECHNOLOGY.
ONE IS THE FAMILY FOUNDATION
THAT FUNDS N F-1 AND THEY HAVE
ENOUGH RESOURCES THAT THEY CAN
ACTUALLY FUND A BROAD ARRAY OF
MOLECULAR TECHNIQUES TO FIND
THERAPY FOR NEURO FIBRO MITO SIS
SO WE WORKED FROM FOUNDATIONS
WHERE OF COURSE YOU KNOW, YOU
HAVE TO BE VERY, VERY CAREFUL
WITH THE EXPERIMENTS ASK MAKE
SURE NOT THAT YOU ARE NOT DOING
MORE THAN ARE NECESSARILY NEEDED
TO GO TOWARDS THE CLINICAL TRIAL
AND ALWAYS OF COURSE MAINTAINING
IN MIND THE SAFETY AND AT LEAST
HAVE A GOOD CHANCE OF HAVING
SOME EFFICACY AND WHATEVER IT IS
THAT BEER DOING SO IT'S A BROAD
EXPERIENCE AND I THINK
DEVELOPING VERY CLOSE
COLLABORATION AND CONTACT WITH
THE FOUNDATIONS AND TO BE
COMPLETELY TRANSPARENT ABOUT THE
GOOD AND THE BAD AND THE
TERRIBLE AND IT'S REALLY
IMPORTANT FOR THE FOUNDATIONS OF
THE PATIENTS SO THAT THEY REALLY
UNDER THE SCIENTIFIC PROGRESS
AND AT THE END OF THE DAY THEY
TRUST WHAT YOU ARE DOING CAN
MAKE A DIFFERENCE AND MOVE THE
FIELD FORWARD FOR PARTICULAR
DISEASES FOR WHICH NOWADAYS WE
DON'T HAVE THERAPIES AND SO I
DID NOT KNOW HOW TO PUT THIS ALL
IN SLIDES SO I JUST GAVE YOU A
FAIRLY LONG MONOLOGUE ON THIS
AND I'M HAPPY TO ANSWER
QUESTIONS LATER ON.
THANK YOU FOR YOUR ATTENTION.
>> THANK YOU.
COULD ALL OF THE SPEAKERS PLEASE
JOIN ME ON VIDEO.
WE HAVE ABOUT 10 MINUTES LEFT
AND I HOPE, I SEE ONE QUESTION
AND I'LL GET US STARTED AND
ENCOURAGE OTHER QUESTIONS TO
COME IN.
I THINK THAT JIG SA METAPHOR
RESINATES WITH MANY OF YOU AND
WHERE A NON-PROFIT CAN HELP
UNDERSTANDING THE LANDSCAPE AND
HELPING ALL THE DIFFERENT TYPES
OF STAKEHOLDERS FIT TOGETHER.
DURING THE OTHER WAYS, ONE THING
THAT I DIDN'T HEAR AS MUCH ABOUT
WAS CONVENING, AND I THINK YOU
MENTIONED THE FAMILY FOUNDATIONS
ANNUAL MEETING, FOR EXAMPLE, AND
HAVE YOU GUYS SEEN EXAMPLES
WHERE YOU FEEL THAT THE NON
PROFITS WORK IN CREATING
SETTINGS TO CONVENE DIFFERENT
TYPES OF STAKEHOLDERS WITHIN
ACADEMIA OR ACROSS ACADEMIA HAS
BEEN PIVOTAL TO THE ADVANCES IN
YOUR FIELD AND I'LL LEAVE IT TO
YOU WHICH OF YOU TO RESPOND?
>> CERTAINLY.
IT'S GOOD TO INVITE ACADEMIC
INVESTIGATORS OF COURSE BUT
COMPANIES THAT MIGHT SHALL
INTERESTED TO COME TO YOUR
ANNUAL MEETINGS.
IT'S BEEN REALLY KAI I THINK
WITH SOME OF THE DISEASES WE
WORK ON TO CONVINCE THE
COMPANIES THAT THERE ARE PEOPLE
WHO REALLY WANT TO HAVE A
TREATMENT AND ARE WILLING TO DO
WHAT THEY CAN TO HELP I THINK IN
PARTICULAR TO SEE WHAT THE
DISEASES ARE LIKE.
I ENCOURAGE THAT FROM PATIENTS
TO INDUSTRY ARE VERY USEFUL.
>> I JUST WANT TO CALL OUT TO
ME, IT RESINATED SO MUCH TO SEE
DIFFERENT STAKEHOLDERS PUT ON TO
EACH OF YOUR DIFFERENT JIG SA
PUZZLE AND I WORK AT THREE
DIFFERENT FOUNDATIONS OR
ASSOCIATIONS AND IT'S SUCH AN
EXAMPLE OF ABOUT HOWDY PENDING
ON THE FIELD OF SCIENCE AND THE
MISSION OF THE ORGANIZATION AND
THE STAKEHOLDERS YOU NEED TO
WORK WITH AND UNDERSTANDING
WHERE THE STAKEHOLDERS ARE
COMING FROM AND BEING EVOLVING
OVER TIME.
WONDERFUL QUESTION IN THE
CHATBOX.
THE FOUNDATION ARE ALTRUISTIC
WANTING TRIALS TO BE SUCCESSFUL,
WHAT IS A FAIR ASK OF FUNDERS OR
NIH OR PHARMA AND INDUSTRY TO
HELP WITH FOUNDATIONS WHO ARE
TRYING TO SUPPORT RECRUITMENT IN
RESEARCH, FOR EXAMPLE.
IS IT REASONABLE FOR FOUNDATIONS
TO MONETIZE THE ACCESS TO
PATIENTS THAT THEY HAVE
CULTIVATED AND I THINK THIS IS A
IMPORTANT QUESTIONS THAT NEEDS
STAKEHOLDERS ANSWERING IT
BECAUSE THERE'S ANSWERS IN
BIOETHICS THAT WE DON'T HAVE IN
THE PANEL RIGHT NOW BUT I WOULD
LOVE TO HEAR THOSE OF YOU FROM
THE ACADEMIC RESEARCH WORLD AND
THE INTRAMURAL RESEARCH WORLD'S.
WHAT DO YOU THINK?
>> WELL, I WAS STARTING TO
RESPONDED IN THE CHAT.
I WOULDN'T PUT ANY BARRIERS TO
RECRUITMENT.
I THINK IT'S GOOD FOR THE
FOUNDATIONS AND MAYBE I'M NOT
READING THIS CORRECTLY.
IT HELPS FOR THE FOUNDATIONS TO
CONVINCE THE ACADEMIC
INVESTIGATORS FIRST AND THE
COMPANIES THAT THEY'RE GUNG HO
GETTING AN EFFECTIVE TREATMENT
AND WILLING TO DO WHAT THEY CAN
WITHOUT CHARGING FOR IT AND
MAYBE I'M READING THIS
BACKWARDS?
>> FOUNDATION NOWADAYS AND
THERE'S A BIG TREND IN THIS IS
THAT IT USED TO BE THAT
FOUNDATIONS ARE PROVIDING
FUNDING AND THAT'S ABOUT IT AND
NOWADAYS MOST FOUNDATIONS WANT
PART OF THE IP THAT IS
GENERATED.
I THINK IT'S COMPLETELY FAIR.
NOW, THE NOTION OF MONETIZING
ACCESS TO PATIENTS, WHILE YOU
PUT A BARRIER THERE AND YOUR
REPRESENTING THE PATIENTS AND IN
MY OPINION, THE REAL OF THE
FOUNDATION AND TO FACILITATE AS
MUCH AS POSSIBLE AND RECRUITING
PATIENTS THAT'S THE GOAL AND THE
PROCESS THEY CAN HAVE THE
INDUSTRY SUPPORT NATURAL HISTORY
STUDIES AND IT'S FABULOUS AND I
PERSONALLY DON'T THINK THAT
CHARGING COMPANIES TO GET ACCESS
TO A LIST OF PATIENTS IS
SOMETHING THAT IT WILL WORK
BECAUSE COMPANIES WILL BE ABLE
TO GET AROUND THAT SO IT FALLS
OUT OF THE MISSION OF A
FOUNDATION TO DO SO AND IT'S A
WAY TO RAISE MUND FOR THE
FOUNDATION BUT THERE'S OTHER
WAYS IT CAN BE DONE SO RESHAPING
HAVE A DIM VIEW OF THAT.
>> I'M HAPPY TO MAKE A COMMENT
ALSO.
I KNOW PAUL WELL AND I'VE HAD
CONVERSATIONS WITH THIS IN THE
PAST AND ONE OF THE GROUPS WITH
PAUL, ONE OF THE GROUPS THAT WE
COLLECT DATA AND IT'S GOING ON
IN A PERSON WAY FOR YEARS AND
FIREFIGHTERS CONSISTENT WITH
BEING ABLE TO DO THAT AND IN
OTHER WORDS DO WE HAVE TO
RECONSENT OR THINK ABOUT IT IN A
DIFFERENT ETHICAL WAY AND IT'S
AN 'EM PEDESTRIANMENT WITH A
NETWORK OR THAT YOU ALL WORK
WITH.
>> WE'VE ANOTHER QUESTION COME
IN FROM KAITLYN AND I'M GOING TO
PARAPHRASE 2 AND I HOPE I DO IT
JUSTICE, KAITLYN, ARE ROLES
AROUND THE NON-PROFIT AND THE
ACADEMIC RESEARCHERS AND
SOMETIMES THE NON-PROFIT HAS
CONTRIBUTION OF SCIENTIFIC
SUPPORT LIKE STUDY DESIGN,
ANALYSIS AND DISSEMINATION AND
I'VE SEEN OTHER NON PROFITS
CURATE DATABASE IS OR INVEST IN
DO YOU HAVE A DEEPER
RELATIONSHIP WITH SCIENTISTS AT
DIFFERENT NON PROFITS?
TO BE HONEST, THERE ARE SO MANY
EXAMPLES THAT I'M HAVING A HARD
TIME COMING UP WITH JUST A FEW
BUT JUST TO START, WITH THE
MAJOR SOME OF THE MAJOR
ORGANIZATIONS AND THERE'S MANY
ALS GROUPS AND NON PROFITS THAT
HAVE ENGAGE WITH RESEARCHERS AND
ACADEMIC CENTERS AND AND THE ALS
ASSOCIATION HAS BEEN FUNDING
MANY COMPLEX RESEARCH STUDIES
AND I MENTIONED A FEW WHEN I
PRESENTED THIS AND THERE ARE SO
MANY MORE AND YOU MAY RECALL AND
THE CHALLENGE AND THAT WAS GREAT
OPPORTUNITY FOR THE ALS
ASSOCIATION TO HAVE MORE FUNDS
AND IT'S PHENOMENAL HOW THEY
DISTRIBUTED THAT TO ALLOW
IMPORTANT RESEARCH TO HAPPEN AND
AGAIN, JUST ONE OF THE MANY
EXAMPLES THAT COULD MAKE BASED
ON ALS ASSOCIATION FUNDING AND
MANY NEW JEANS FOR ALS HAS BEEN
DISCOVERED BECAUSE OF THE
FUNDING THAT THEY DISTRIBUTE SOD
CLEARLY ASSOCIATIONS LIKE THIS
ONE LIKE THE ASSOCIATION HELPED
NOT JUST FOR RECRUITMENT OR
COLLABORATION WITH THE PATIENT
COMMUNITY BUT TO ACTUALLY FUND
RESEARCH THAT HAS PROVEN TO BE
TRANSFORMATIVE AND IT'S ONE OF
THE EXAMPLES AND OTHER
ASSOCIATIONS LIKE FOR EXAMPLE
THE MDA OR MUSCULAR DYSTROPHY
ASSOCIATION HAVE A MOVING
REGISTRY THAT HELPS AMOUNT IN
TERMS 6 KNEW OWE MUSCULAR
DISEASE AND PROVIDES
INFRASTRUCTURE FOR RESEARCH FOR
NATURAL HISTORY STUD EYES AND IT
COULD GO ON AND ON SO ESSENTIAL
LOW THERE'S SEVERAL EXAMPLES AND
I WOULD LIKE TO EMPHASIZE THE
IMPORTANCE OF THIS RESEARCH
SOMETIMES FOUNDATIONS FUND
HIGH-RISK AND HIGH REWARD
PROJECTS THAT OTHERWISE WOULD
TAKE A LONG TIME TO GET FUNDED
THROUGH MORE TRADITIONAL SOURCES
SO THEY HE CAN SELL RATE THE
PROGRESS.
>> ONE MINUTE WARNING.
>> YOU ARE MUTED.
>> ONE EXPERIENCE TO JUST
DESCRIBE REALLY BRIEFLY IS
CONVINCING BIOGEN TO TAKE ON
SPINAL AND I WENT THERE WITH BY
OUR INSTITUTE DIRECTOR AT THE
TIME AND SAY COUPLE FOUNDATION
AND HAVE I WELL-INFORMED COUPLE
AND GETTING OUT TO BUY ONTO THE
IDEA AND WORKING ON A RELATIVELY
RARE DISEASE LIKE SPINAL
MUSCULAR ATROPHY IS GETTING
THERAPEUTICS GOING FOR THAT
DISEASE AND SO IT WAS A REAL
TURNING POINT AND IT WAS FUN TO
BE THERE FOR THAT.
>> IT WAS ASPIRING.
WE'RE ALMOST OUT OF TIME.
>> I THINK THE INCOMPETENTER
ACTION WITH THE PATIENTS OF
MULTIPLE IS IMPORTANT AS A
REALITY CHECK BECAUSE SOMETIMES
CERTAIN TESTS AND FREQUENCIES
ARE PERFECTLY LOGICAL AND GIVEN
THE CONDITIONS OF PATIENTS OFTEN
THAT LEADS TO VERY HIGH RATES OF
ATTRITION AND CLIP CAL TRAILS
AND SO IT'S IMPORTANT TO HAVE
THE SENSE OF WHAT PATIENTS ARE
LIKELY TO DO AND WHAT WOULD LIKE
TO BE DONE AND THOSE TWO COULD
BE VERY DIFFERENT.
>> IT WAS REALLY WONDERFUL TO
HEAR FROM ALL OF YOU WITH ALL OF
YOUR REMARKS.
WE ARE AT TIME.
IT'S BEEN AN HONOR TO HAVE YOU
WITH US TODAY AND AN INSPIRATION
TO HEAR THE SUCCESS STORIES
YOU'VE BEEN ABLE TO SHARE.
>> THANK YOU.
THANK YOU.
>> THANK YOU ALL.
THANK YOU ALL OF OUR SPEAKERS
FOR THAT AMAZING SESSION.
SO, I AM GOING TO TAKE US INTO
DAY ONE AND THE MEETING BEFORE I
DO THAT AND I WANT TO REMIND
EVERYONE THERE'S AN OPPORTUNITY
TO SCHEDULE ONE-ON-ONE MEETINGS
WITH NIH STAFF WE'VE BEEN
PLACING THE LENGTH IN THE CHAT
AND YOU CAN CHECK YOUR E-MAIL IF
YOU ARE REGISTERED FOR THE EVENT
AND IF YOU ARE NOT REGISTERED,
YOU CAN CERTAINLY STILL REGISTER
NOW OR TOMORROW AND ADDITIONALLY
TOMORROW AVERAGE, THERE'S
BREAKOUT SESSIONS FOR NON NIH
PARTICIPANTS OF THE MEETING AND
THERE'S A SURVEY THAT WE WOULD
LOVE FOR AWFUL YOU TO FILL OUT
TO HELP US FIGURE OUT TO BREAK
OUT ROOMS SO WITHOUT FURTHER
ADIEU OUR NEXT SESSION IS TITLED
ENGAGING TRAINEES IN EARLY STAGE
SCIENTISTS HOW TO WORK WITH
ACADEMIC RESEARCHERS AND THESE
ARE KIND OF LIGHTENING TALKS,
IT'S A LITTLE BIT MORE OF A
ROUND-ROBIN FIRESIDE CHAT WE'RE
GOING TO AND WE'RE DELIGHTED TO
HAVE Dr. NINA SCHOR MODERATE
THE SESSION AND NINA IS THE
SCIENTIFIC DIRECTOR OF NINDS
BEFORE COMING TO NIH, SHE SPENT
20 YEARS ON FACULTY AT THE
UNIVERSITY OF PITTSBURGH
BECOMING THE CAROLE PROFESSOR
THE PEDIATRIC RESEARCH AND THE
CHIEF OF DIVISION OF CHILD
NEUROLOGY AND THE DEPARTMENT OF
PEDIATRIC AND DEAN FOR MEDICAL
RESEARCH AND THE MEDICAL SCHOOL
AND IN 2006, THE CHAIR OF THE
AND AND AT UNIVERSITY OF
ROCHESTER AND CHILD IN
JANUARY 2018 AT WHICH POINT SHE
CAME TO AND FOR 27 YEARS IT WAS
CONTINUOUSLY FUNDED BY NIH AND
SHE CURRENTLY LEADS THE DIVISION
OF INTRAMURAL RESEARCH AND THE
ULTRARARE GENE TARGETING THERAPY
OR URGENT NETWORK AND SHE'S ALSO
DONE TONS OF WORK FOR NINDS ON
OUR PLAN FOR THE NEXT FEW YEARS
AS WELL AS CAREER DEVELOPMENT
PROGRAMS AT NINDS AND SHE SERVES
AS A NEUROLOGY DIRECTOR FOR THE
AMERICAN BOARD OF PSYCHIATRY AND
ONCE WE HIT AUGUST, Dr. SHORE
WILL MOVE ON AND UP IN NIH TO BE
THE DEPUTY DIRECTOR FOR ENTIRE
TEE OF NIH SO WITH THAT I WOULD
LIKE TO PASS THE TORCH TO Dr.
SHORE SO SHE CAN PICK US OFF
WITH THIS NEXT SESSION.
THANK YOU INFORM ALLFUL YOU FOR
JOINING US THIS AFTERNOON AND I
KNOW TIME IS JUST VERY THE
SESSION THAT FOLLOWS WILL BE ON
NOMINALLY ENGAGING TRAINEES AND
EARLY STAGE SCIENTISTS BUT I'M
HOPING WE GET A DISCUSSION GOING
WELL BEYOND THAT AND THAT
ENGAGES ALL OF YOU BECAUSE THIS
IS A ROLE AND FUNCTION THAT NON
PROFITS PLAY NOT ONLY FOR THE
SCIENTIFIC COMMUNITY BUT
ESPECIALLY FOR THE PATIENTS AND
FAMILIES THAT THEY SERVE, ONE OF
THE MOST CHALLENGING TIMES IN A
SCIENTIST LIFE IS AT THE
BEGINNING, HOW DO YOU CONVINCE
ANYBODY THAT YOU ARE HOPING TO
ANSWER AN IMPORTANT QUESTION AND
IMPACTFUL QUESTION AND HOW DO
YOU CONVINCE ANYBODY THAT YOU,
WHOSE NAME IS NOT YET KNOWN, TO
THE SCIENTIFIC COMMUNITY, THAT
YOU ARE THE BEST PERSON TO DO
THAT WORK AND I THINK MOST OF US
AT THAT STAGE IN OUR CAREERS
HAVE DEPENDED IN VERY LARGE
MEASURE ON FUNDING FROM VERY
DEDICATED NON-PROFIT
ORGANIZATION AND THERE'S ANOTHER
TIME IN A SCIENTIST LIFE THAT IS
CHALLENGING AS WELL AND THAT IS
RIGHT AROUND MID CAREER WHEN
YOU'VE HAD YOUR INDEPENDENT
GRANT FUNDING PERHAPS A FEDERAL
AGENCY OR LARGER NON PRIVILEGE
ITS AND YOU LOOK TO RENEW IT AND
AGAIN, THEY CAN PLAY A ROLE TO
HELP WITH PRELIMINARY D FOR THE
RENEWAL OF A GRANT WITH HELPING
TO DIRECT INDIVIDUALS IN A
DIRECTION THAT IS MOST HELPFUL,
IMPACTFUL, MEANING TO TO THE
LIVES OF PATIENTS AND FAMILIES
SO TO ADDRESS THIS WE HAVE FOUR
SPEAKERS TODAY.
THEY'RE EACH GOING TO GIVE
RELATIVELY BRIEF TALKS AND NONE
OF THEM HAVE SLIDES SO THIS WILL
BE VERY IMPROMPTU KIND OF A
DISCUSSION TWO OF OUR SPEAKERS
ARE FROM ACADEMIA AND TWO ARE
FROM NON PROFITS AND I'M GOING
TO INTEREST THEM EACH BEFORE
THEIR TALKS AND OUR FIRST
SPEAKER IS Dr. DANIEL OKO BYE.
HE GOT HIS PH.D IN NEUROSCIENCE
AT NYU AND HIS MD ALSO FROM NYU
AND HIS CLINICAL TRAINING HE DID
AT THE UNIVERSITY OF MIAMI AND
AT UCLA AND HE STAYED ON AT UCLA
WHERE HE IS NOW FACULTY AND
SPECIALIZES IN BEHAVIORAL
NURROLOGY SO I WILL HAND THE
FLOOR OVER TO Dr. OKOBI.
>> GOOD AFTERNOON.
I'M A BEHAVIORAL NEUROLOGY
FELLOW AND CLINICAL STRICT OR
AND JUNIOR ATTENDING IN SOME WAY
AND I WORK WITH THE GROUP THAT
WORKS ON DIFFERENT TYPES OF
NEUROLOGICAL DISEASES AND
TRADITIONALLY IT'S BEEN AT OF
GENETIC BASED AWE AT THE SAME
TIME AND EPILEPSIES AND I WANTED
TO CONTINUE A LITTLE BIT TOWARDS
DISEASE THAT A LOT OF PEOPLE
HAVE BEEN THINKING ABOUT AND
IT'S DEMENTIA AND WHEN I CAME
OVER, TO WORK IN RESIDENCY AND I
TALKED TO THE CHAIR AND I TALKED
ABOUT LOOKING AT THE PROJECTS
ABOUT WHAT KIND OF THE BEST
QUESTIONS TO ANSWER AND WHAT THE
FIELD IS KIND OF LOOKING FOR
BECAUSE I ACTUALLY WORKED ON
BEHAVIOR AND I HAD NOT REALLY
THOUGHT ABOUT BEHAVIORAL
NEUROLOGY AND BASICALLY,
DEVELOPMENTAL DISABILITY IN THE
ADULT POPULATION IT'S DEMENTIA
AND NEURO PSYCHIATRY AND I HAD
BEEN A MEMBER OF THE SOCIETY FOR
NEUROSCIENCE AND I WANTED TO
ATTACK IT TO LOOK AT THE
CIRCUITRY AND DEMENTIA WHICH NOT
HAS MANY PEOPLE HAVE AND MY IDEA
OF LOOKING AT THE DEMENTIA WAS
LOOKING AT NOT EVERYONE IS
ACTUALLY LOOKING INTO SO ONE OF
THE DEMENTIAS I WAS INTERESTED
IN IS AT UCLA WE HAVE A
SPECIALIST EXPERT THAT RUNS A
FELLOWSHIP I'M A PART OF AND FOR
TEMPORAL DEMENTIA IS MORE
GENETIC THAN THE AVERAGE
DEMENTIA AN AND IT TENDS TO BE E
BEHAVIORAL VANT WHICH IS WHEN
PEOPLE NOTICE THAT THEIR LOVED
ONES CAN BE THEY MIGHT EATING
SWEETS AND THEY MIGHT START TO
GET VERY SEXUALLY FRANK IF THEY
WERE LIKE A RESERVED PERSON SO
LIKE A CHANGE IN-PERSON A LOT
AND JUST BEHAVIORAL AUD TEES AND
THAT PRE DATES WHAT WE THINK
ABOUT DEMENTIA WHICH IS MEMORY
ISSUES WHERE PEOPLE HAVE
LANGUAGE ISSUES AND TO BE ABLE
TO COMMUNICATE IS TO THOSE ARE
THE TWO TYPES OF DEMENTIA AND
WHAT I'M BUILDING ON IS THE A
LOT OF PEOPLE HAVE BEEN WORKING
ON THIS AND IN SOME RESPECT SO A
LOT OF PEOPLE HAVE BEEN WORKING
ON THE GENETICS AND
UNDERSTANDING HOW THIS IS IN THE
BRAIN CAUSES LOTS OF DAMAGE AND
I WANT TO BE ONE OF THE PEOPLE O
TO UNDERSTAND IT IS AS THE
DEMENTIA IS RAIN BECAUSE WE
ACTUALLY CAN RESTRAIN CIRCUITS
AND PEOPLE HAVE BEEN LOOK AT SO
I WANT TO SEE ALSO THERE'S
SOMETHING THERE IN TERMS OF THE
ACTUAL CIRCUITRY BEING BROKEN
DOWN SO I CAN AT LEAST ESTABLISH
THAT IF WE TROY TREATMENTS OF
ACTUAL LOW CAN REWIRE OR FIX
WHAT IS BEING BROKE EASTBOUND
DOWN AS THE D DEMENTIA IS HELPIG
IT'S A WAY TO BRING DECELERATION
TO THE PATTERN OF DEMENTIA AND
THE THING THAN IS NOT UNIQUE TO
THE FRONTAL TEMPORAL DEMENTIA IS
THERE IS NOT REALLY A TREATMENT
THAT IS REVERSING THE
FUNDAMENTAL DAMAGE THAT IS
HAPPENING YET AND AS WE HEARD
EARLIER ABOUT FDA APPROVALS FOR
MEDICATIONS JUST BECAUSE THE FD
APPROVALS THE MEDICATION DOESN'T
MEAN THAT EVERYONE WHO NEEDS IT
OR GETS ACCESSIBLE THERAPEUTIC
RESOLUTION SO WE NEED TO DO A
LOT OF DIFFERENT THINGS 245 WE
DON'T KNOW WHAT WILL WORK BUT IF
WE FIND SOMETHING, WE SHOULD TRY
IT.
SO I'LL STOP THERE BECAUSE I
KNOW THERE ARE OTHER PEOPLE AND
YOU MIGHT BE ON MUTE.
>> THANK YOU AND OUR NEXT
SPEAKER IS Dr. ERIC MORROW.
Dr. MORROW GOT HIS PH.D IN
GENETICS AND NEURO DEVELOPMENT
AT HARVARD AND OF MOLECULAR CELL
BIOLOGY AND Dr. MORROW.
>> THANK YOU, VERY MUCH, Dr.
SCHOR.
WE GET THUMBS UP, I'M THRILLED
TO BE PARTICIPATING IN THIS
CONFERENCE AND I'VE ENJOYED THE
SESSIONS.
SO AS Dr. SCHOR SAID, I'M AT
BROWN UNIVERSITY AND I'M A
PHYSICIAN SCIENTIST AND I WORK
IN A BASIC SCIENCE DEPARTMENT
BUT I ALSO WORK IN CLINICAL
DEPARTMENTS WHERE YOU DO
RESEARCH WITH FAMILIES WITH
NEURO GENETIC DISORDERS AND IT'S
MY CLINICAL RESEARCH SO THE
PATIENTS ORIENTED WORK WHERE
I'VE COME INTO CONTACT WITH
DIFFERENT FAMILY-LED NON PROFITS
SO ONE GENETIC DISORDER IS
CHRISTIANSON SYNDROME AND WE
NAMED IT AFTER THE SPEED TRICK
AN WHO NEWER LOGIC DISORDERS
CAUSES DEVELOPMENTAL DISABILITY
AND AS WELL AS NEURODEGENERATIVE
FEATURES AND I WAS WORKING ON
THIS CONDITION IN MY BASIC
SCIENCE LAB AND HAD NOT STARTED
WORKING WITH THE FAMILIES AND I
GOT AN E-MAIL OF AN EFFECTED,
FROM THE MOTHER OF A EFFECTED
PATIENTS PERSON, MALE WITH THIS
CONTINUE AND SAYING THAT SHE
JUST GOT THE GENETIC DIAGNOSIS
AND SHE DOESN'T KNOW ABOUT THE
CONDITION BUT THEY READ A PAPER
FROM READING THE INTERNET AND
WHAT I SPEAK WITH HER ABOUT IT
AND THIS WAS 2010 OR 2011 AND
HER NAME WAS DEBBIE NASH AND
I'VE HAD THE HONOR OF WORKING
WITH DEBBIE WHO FOUNDED THE
CHRISTIANSON SYNDROME
ASSOCIATION AND IT'S A
NON-PROFIT TO RAISE AWARENESS
ABOUT CHRISTIANSON SYNDROME AND
SUPPORT RESEARCH AND SO I'VE HAD
THE HONOR AND PRIVILEGE OF
WORKING WITH FAMILIES WHO RUN
NON PROFITS FROM THE VERY EARLY
STAGES AND THE FOUNDING OF THE
KRISTEN SON FOUNDATION WENT LIKE
THIS AND DEBBIE HAVE YOU MET
OTHER FAMILIES AND ONE OF THE
FAMILIES STARTED A Facebook
GROUP AND WOAD OFFERED AT BROWN
UNIVERSITY TO DONATE SPACE OR TO
THE FIRST CONFERENCE CAME
TOGETHER AT THAT FIRST MEETING
IN 2013 AND AT BROWN UNIVERSITY
AND THE RESEARCH HAS GROWN AND
DEVELOPED AND IN MY GROUP AND IN
OTHER SCIENTIFIC OVER THE LAST
DECADE AND IN 2015 AND WE WERE
FORTUNATE TO GET THEY HAVEN'T
MET FAMILIES THAT ARE EFFECTED
AND THESE CONFERENCES ARE
WONDERFUL TO HAVE THE
OPPORTUNITY TO BRING THEY'RE
MORE AND MORE CLINICAL AND MORE
AND MORE INTERNATIONAL AND
THERE'S AN INTERNATIONAL AND
THERE'S A SURE PEE AN WHERE TIVE
FAMILIES AND LANGUAGES AND AND
ONE POINT THAT I WILL MAKE THAT
I THINK HAS BEEN MADE AND ON
TRAINEES AND ALSO FOR HELPING
THE SO FOR EXAMPLE AND TO
CHRISTIANSON DIN DROP WE'VE
RECEIVED FUNDING AND
CHRISTIANSON AND AND LARGER ONE
SUCH AS THE OUR WORK OF OTHER
PEOPLE IN THE WORLD THE MORE
ESTABLISHED NON PROFITS AND
AUTISM AND EP DEPOSIT SEE AND
LARGER AREAS WHERE THE RARE
GENETIC DISORDERS COME TOGETHER
AND REALLY CRITICAL AND SO I
THINK THAT RELATIONSHIP HAS BEEN
CRITICAL AND BEFORE I WRAP-UP IS
THERE HAVE BEEN I THINK SOME
VERY, VERY INTERESTING FUNDING
GRANTS AND MAYBE WE CAN DISCUSS
THIS IN THE DISCUSSION PERIOD
AND FROM NEW FOUNDATION AND
MONDAY PROFITS THAT REPRESENT
COP AGO RACES AND BETWEEN THE
NON PROFITS AND THE RESEA RESEAS
THAT WOVE HAD PLIED FOR IS THE
ZUCKERBERG INSTITUTE HAS FUNDED
A SERIES OF GRANTS AND THE AREA
OF RARE GENETIC DISORDERS ONE
CALLED RARE IS ONE AND THE
SECOND ONE LAST MAY AND ON THE
NON PROFITS ARE OWE ON CANNES
TOGETHER ON A SCIENTIFIC
ENDEAVOR WHICH REALLY ORGANIZES
THE COLLABORATION BETWEEN THE
ACADEMIC RESEARCHERS AND THE
NON-PROFIT IN THE APPLICATIONS I
THINK THESE CONFERENCES IN THE
EXCHANGE WHETHER IT'S E-MAIL,
SOCIAL MEDIA, ARE CRITICAL IT'S
A TWO-WAY STREET AND INSPIRING
COLLABORATION AND THE
RELATIONSHIPS BETWEEN THE EARLY
FORMING NON PROFITS AND THE MORE
ESTABLISHED IS I THINK THE THEME
IS WE HAVE A LOT TO LEARN FROM
EACH OTHER AND THANK YOU.
>> THANK YOU, Dr. MORROW.
OUR NEXT SPEAKER IS Dr. DIANNE
BOVENKAMP.
SHE GOT HER PH.D FROM QUEEN'S
UNIVERSITY IN KING STON,
ONTARIO, AND WENT ON TO DO A
POSTDOC TO BAL FELLOWSHIP.
SHE'S VICE PRESIDENT FOR
SCIENTIFIC AFFAIRS AT BRIGHT
FOCUS FOUNDATION.
Dr. BOVENKAMP.
>> GREAT, CAN EVERYBODY HEAR ME
BECAUSE I WAS TOLD MY VOLUME WAS
LOW BEFORE?
>> SOUNDS GOOD.
>> OK.
SO I'M WITH BRIGHTFOCUS, 12
YEARS NOW IN VARIOUS CAPACITY
BUT RIGHT NOW I'M ALMOST ALL
ASPECTS OF OUR GRANTING.
LIKE OTHER ORGANIZATIONS, THAT
WE -- OTHER NON PROFITS, THE
SCIENCE HELPS WITH THE
FUNDRAISING AS WELL AS THE
COMMUNICATIONS AS WELL AND SO
WE'VE BEEN NEXT YEAR IS GOING TO
BE OUR 50th ANNIVERSARY
ACTUALLY AND WE USED TO BE
CALLED AMERICAN HEALTH
ASSISTANCE FOUNDATION AND WE
REBRANDED IN 2013 SO SOME OF YOU
WHO HAVE HAD AN AWARD THAT WAS
US AND SO YEAH SO WE'VE BEEN
AROUND A WHILE AND YOU KNOW, FOR
THE LAST TWO YEARS, WE'VE GIVEN
$25 MILLION IN GRANTS EACH YEAR
IN OUR THREE PROGRAMS SO WE FUND
ALZHEIMER'S DISEASE, GLAUCOMA
AND MACULAR DEGENERATION AND
WHAT'S THE CONNECTION?
SO WHEN WE STARTED OUT SMALL IN
1973, IT WAS REALLY THE THING
THAT BINDS IT ALL TOGETHER IS
THEIR DISEASES OF AGING, RIGHT.
AND A LOT OF PEOPLE GET THEM
WHEN THEY GET OLDER AND A LOT OF
THE FOUNDERS FAMILY MEMBERS HAD
THEM AND NOW ACTUALLY THERE'S A
LOT OF SCIENTIFIC TIES SO
THEY'RE ALL GENERATIVE DISEASES
AND AT THE FOUNDATION WE HAVE
OUR OFFICIAL JOURNAL IS
MOLECULAR DEGENERATION AND A LOT
OF PUBLICATIONS THERE WITH BOTH
THE VISION AND THE BRAIN
CONNECTION THAT WE FUND.
WE FUND RESEARCH AS WELL AS
WE'RE CONNECTIONING WITH I THINK
IT'S ABOUT 500,000 PEOPLE ACROSS
THE U.S. AND BEYOND BECAUSE
WE'RE AN INTERNATIONAL FUNDER
AND WE GIVE NOT ONLY GRANTS TO
SCIENTISTS AND OTHER NON
MONETARY SUPPORT AS I'LL JUST
BRIEFLY ALIGN LATER, BUT WE ALSO
COMMUNICATE SCIENCE TO
INDIVIDUALS AND ACTUALLY WE HAVR
COMMUNICATIONS OF ALL OF THE
RESEARCH THAT WE FUND AND THE
BIG ACHIEVEMENTS IN THE FIELD TO
EFFECTED FAMILIES AND WE HAVE A
1-800 NUMBER SO WE STRADDLE THE
LINE BETWEEN I WOULD SAY THERE'S
FOUR WAYS THAT I'LL BREAK IT
DOWN THAT WE ENGAGE TRAININGS IN
EARLY STAGE SCIENTISTS AND I
KNOW THAT EVERY NON-PROFIT IS
DIFFERENT AND SOME PEOPLE DO
MORE AND ONE OF THE FINANCIAL SO
WE HAVE GRANTS IF ALL THROW
PROGRAMS AND FELLOWSHIPS AND ALL
OF THEM AND PEOPLE WHO ARE WE
HAVE NEW INVESTIGATOR AWARDS AS
WELL AS SEASONED INDIVIDUALS
AWARDS OR STANDARDS AWARDS SO WE
FUND THE WHOLE GAMUT AND WE ARE
MORE OF A TRADITIONAL, WE TEND
TO NOT NECESSARILY SAND OUT
SPECIFIC IDEAS OR SPECIFIC
RFPs IT'S INVESTIGATOR
INITIATED AND THIS IS THIS COULD
BE WHERE WE WE ALSO REALLY TRY
AND ENCOURAGE PEOPLE LIKE DANIEL
TO COME IN AND WHO ARE EXPERTS
IN ONE FIELD TO COME IN AND
APPLY AND LIKE WITH ERIC WE
ACTUALLY DO AGREE WE DO FUND
IT'S GOOD FOR PEOPLE TO COME IN
AND GET FUNDING TO SHOW THE
CROSS OVER BETWEEN ALZHEIMER'S
AND OTHER DEMENTIA OR MAYBE A
RARE FORM OF MACULAR
DEGENERATION SO PEOPLE SHOULD
TRY AND AND WE'VE PARTNERED WITH
OTHER ORGANIZATIONS BEFORE TOO
SO YOU ARE HITTING ALL THE OF
THE -- HOW DO YOU FIND THE
FOUNDATION THAT IS IMPORTANT AND
SO WE ACTUALLY DO HEALTH WITH NO
HOUSE AND SOME OF THE OTHER
THINGS THAT WE DO.
WE HAVE SIGNATURE CONFERENCES WE
CALL FAST TRACK AND IT'S A
BOOTCAMP AND HEAR FROM
INVESTIGATORS IN THE FIELD AND
NETWORK WITH THEIR COLLEAGUES
AND WITH SEASONED INDIVIDUALS
AND AS WELL AS WE HAVE BREAKFAST
AT MAJOR CONFERENCES WHERE
PEOPLE CAN NETWORK AND WEBINARS
AND THOSE ARE LEARNING HOW TO
CONNECT.
THERE'S OTHER WAYS THAT WE CAN
HELP TO CONNECT TOO SO A LOT OF
PEOPLE WHILE TRYING TO MAKE
CONNECTIONS IN THEIR FIELD IT
MIGHT NOT THINK ABOUT THE
NON-PROFIT GRANTERS BUT NOT ONLY
CAN PEOPLE CONNECT WITH US FOR
FEEDBACK ON SPECIFIC GAINS WHEN
THEY'RE APPLYING BUT ALSO WE
KNOW A LOT OF PEOPLE IN THE FOR
PROFIT AND OTHER NON PROFITS SO
WE CAN INTRODUCE YOU TO OTHERS
OR MAYBE WE MIGHT BE ABLE TO
TELL YOU, YOU KNOW, HAVE YOU
CONSIDERED APPLYING TO THIS
ORGANIZATION SO IT'S A GOOD WAY
TO TRY AND CONNECT AND I THINK
THAT THE FINAL THING SO THERE'S
THE NO HOW AND THE FINANCIAL AND
THERE'S THE THE
FOURTH WOULD BE WE HAVE AN
ALLUMNI NETWORK SO DON'T STOP
THINKING ABOUT THE NON-PROFIT
ORGANIZATIONS WHEN THE FUNDING
ENDS.
WHEN WE'VE FUNDED YOU, A LOT OF
TIMES WE'RE YOUR BIGGEST
CHAMPIONS AND SO WE HAVE THINGS
AND I THINK THAT THERE ARE SOME
DOCUMENTS THAT I DID SHARE
ASPECTASEXAMPLES.
WE HAVE YEAR BOOKS WITH PEOPLE'S
MUGSHOTS WITH DESCRIPTIONS AND
YEAR BOOKS AND ANNUAL REPORTS
AND RESEARCH AND BRIEF AND WE'LL
BRING PEOPLE IN FOR TALKS AND
TALK WITH MAJOR DONORS SO AND
INTRODUCE YOU TO PEOPLE IN THE
FIELD SO THERE'S A LOT MORE BUT
WE SHOULD GET ON TO HAVING
JENNIFER CHAT AND WE CAN HAVE
DISCUSSIONS.
>> GREAT, THANK YOU.
AND OUR FINAL SPEAKER IS
JENNIFER BRUMMET.
SHE GOT HER BACHELORS DISEASE IN
PSYCHOLOGY AT THE ILLINOIS
WESLEY ANNE UNIVERSITY.
HER PH.D IN PSYCHOLOGY AND
BEHAVIORAL NEUROSCIENCE AT
MICHIGAN STATE AND WORKED IN
GOVERNMENT AGENCIES AND
ORGANIZATIONS AND SOCIETY OF
NEURAL SCIENCE, NSF, THE
AMERICAN BAR FOUNDATION AND
SHE'S CURRENT LEE THE MANAGER
FOR RESEARCH AT THE AMERICAN EP
LHEEPILEPSIES SOCIETY.
>> I'M THE CLEAN UP HITTER HERE
TODAY SO I'M GOING TO SPEND A
FEW MINUTES GIVING A HIGH-LEVEL
OVERVIEW OF HOW THE AMERICAN
EPILEPSIES SOCIETY WORKS WITH
EARLY CAREER INVESTIGATORS AND I
WANT TO USE THE LAST PART OF MY
TIME TO KIND OF DISCUSS A FEW
OVERARCHING THEMES AND SOME
LESSON LEARNED BEFORE WE LAUNCH
INTO THE Q&A.
SO FIRST, A QUICK OVERVIEW OF
THE DIFFERENT WAYS THAT WE WORK
WITH EARLY CAREER INVESTIGATORS
AT THE AMERICAN EPILEPSIES
SOCIETY.
I WORK WITH SOME OF OUR
VOLUNTEER COMMITTEES TO SUPPORT
EARLY KA RO CAREER INVESTIGATORS
INCLUDING NETWORKING
OPPORTUNITIES AND WE DO THIS
THROUGH ON-LINE TRAINING MODULES
AND WEBINARS, EPILEPSIES,
SPECIFIC ASSESSMENTS ON-LINE AND
TARGETED CONTENT FOR EARLY
CAREER AUDIENCE AND OUR ANNUAL
SCIENTIFIC CONFERENCE AND
INCLUDING CAREER PATH WAY
SESSIONS AND ADDITIONAL
INFORMATION SESSIONS TO HELP ON
BOARD THEM TO THE SOCIETY IN OUR
COMMUNITY.
ONE OF OUR FLAGSHIP PROGRAMS
THAT WE OFFER IS CALLED THE
FELLOWS PROGRAM AND THOSE
PROGRAMS PROVIDES SUPPORT FOR
CLINICAL FELLOWS AND POSTDOC TO
BAL RESEARCH FELLOWS TO ATTEND
OUR ANNUAL SCIENTIFIC CONFERENCE
AND DURING THIS PERIOD AT OUR
MEETING, THEY'LL GET
PROFESSIONAL DEVELOPMENT CONTENT
AND THEY GET CONNECTED WITH
MENTOR SO REALLY THE GOAL THERE
IS TO PROVIDE THEM THIS
NETWORKING AND ONBOARDING
OPPORTUNITY TO OUR COMMUNITY.
IN ADDITION TO THESE OTHER
OPPORTUNITIES, WE ALSO PROVIDE
GRANT FUNDING WHICH EVERYONE
KNOWS IS CRITICAL FOR EARLY
CAREER SCIENTISTS AND ABOUT 85%
OF OUR GRANT FUNDS GO TO EARLY
CAREER INVESTIGATORS AND
CLINICIAN SCIENTISTS RANGING
FROM THE PH.D STAGE 20 THE AND
OF COURSE OUR GOAL IS TO
MENTORING AND NETWORKING AND
OPPORTUNITIES AND WE HAVE ALUMNI
EVENTS INCLUDING A RECEPTION AT
OUR CON YOU'LL CONFERENCE AND TO
HELP CONNECT SOME OF OUR
GRANTEES WITH OTHER NON-PROFIT
PARTNERS THAT WE WORK WITH THAT
MIGHT FOCUS ON VARIOUS
EPILEPSIESES ARE GENETIC
EPILEPSIES AND THE NINDS PROGRAM
OFFICERS AND THAT'S REALLY PART
OF OUR COMMITMENT TO YOU KNOW,
WE WITHIN TO FUND THEM AND WE
WANT THEM TO HAVE GREAT GRAND
OUTCOMES WHEN SO BECAUSE OF THAT
GOAL AS WELL WE TROY TO HELP
TRAINEES AND LAUNCH THE GRA CORE
AND WE SERVE AS GRANT REVIEWERS
WHICH HELPS US BUT HOPEFULLY
ALSO HELPING THEM CONTINUE TO
DEVELOP THEIR OWN.
SO I'LL USE THE NEXT THREE
MINUTES TO TALK ABOUT SOME OF MY
LESSON LEARNED OVER THE YEARS SO
I'LL BE INTERESTED TO SEE WHAT
THE OTHER PANELISTS AND TO
ENCOURAGE THE NON-PROFIT ON THE
CALL TO REALLY PUT A LOT OF WORK
NO IDENTIFYING THE NEEDS OF YOUR
COMMUNITY AND IT'S REALLY
IMPORTANT TO UNDERSTAND THEIR
TRADING ENVIRONMENTS AND CHARGES
FACE AND CAREER TRAJECTORY AND
ABOUT WHAT RESOURCES THEY NEED
TO SUITE AND THIS CHANGES OVER
TIME, RIGHT.
SO THIS ISN'T NECESSARY ONE OFF
TASK AND YOU KNOW THE FUNDING
NEEDS MIGHT CHANGE OVER TIME.
AND IN ADDITION TOP
UNDERSTANDING THE NEEDS OF YOUR
COMMUNITY, I WOULD ALSO PUT ONE
POINT OUT THERE WHICH IS TO
UNDERSTAND WHAT THE EARLY KA
RAILROAD INVESTIGATORS WOULD
LOOK TO YOUR ORGANIZATION FOR.
NONE OF US IS NON PROFITS HAVE
UNLIMITED CAPACITY OR FUNDS SO
IT'S JUST REALLY HELPFUL TO MAKE
SURE ONE EXAMPLE IS WE HAVE TO
PUT A LOT OF IT WAS WELL ALIGNED
WITH THE NEEDS OF OUR COMMUNITY
AND UNDER UTILIZED SO WITH A
LITTLE BIT DIGGING REAL REALIZED
WHAT WE WERE OFFERING WAS
OFFERED BY THE INSTITUTIONS AND
SO WHAT T ALLOWED US TO TAKE A
STEP BACK AND WORK TO REFINE OUR
PROGRAM IN A WAY THAT REALLY
HONED IN ON OR ORGANIZATION
UNIQUE EX PER HE'S AND REALLY
HELPED GET THOSE RESOURCES OUT
THERE TO OUR EARLY CAREER
INVESTIGATORS.
AND THE SECOND BIG LESSON IS TO
IDENTIFY WAYS TO MAKE WORKING
WITH EARLY CAREER INVESTIGATORS
MUTUALLY BENEFICIAL AND IT
DOESN'T NEED TO BE EQUALLY
BENEFICIAL BUT I THINK THE BEST
RELATIONSHIP TEND TO BRING
SOMETHING OF BENEFIT TO BOTH
SIDES AND SO EVEN A GRANT
PROGRAM CAN BE BENEFICIAL RIGHT
AND OBVIOUSLY EARLY CAREER
INVESTIGATORS AND HOCH YOU CAN
TORCHBEARER TO PULL IN YOUR PAST
GRANT TEES AS A VOLUNTEER FOR
OTHER PROGRAMS OR A GRANT
REVIEWERS AND OR BUILD AN
ONGOING COMMUNITY AND PEOPLE
WORKING ON A SPECIFIC DISEASE
AREA OR REALLY ANYTHING OF THEY
HAVE HAD A LOT OF CHANG HES
FUNDING AND RESEARCH AND THE SO
IN MY OPINION THE POSITIVE SPIN
ON THIS IS THERE'S A LOT OF
FUNDS ARE NOT PROFIT TO STEP IN
AND IDENTIFY SOME EMERGING NEEDS
AND OPPORTUNITIES TO WORK WITH
TRAINEES WHETHER IT'S FUNDING
PROGRAMS AND OR PROVIDING OTHER
RESEARCHES AND SO I WILL END
THERE AND I LOOK FORWARD TO THE
Q&A.
>> THANK YOU.
SO, THIS IS YOUR TIME, YOU ARE
AUDIENCE TO ASK US ANYTHING YOU
WANT AND TO PERHAPS SUGGESTING
THINGS THAT YOU WOULD LIKE TO
HEAR DISCUSSION ABOUT.
I'M HAPPY TO GET THINGS STARTED
BECAUSE AS I HEARD EACH OF THE
TALKS THE THING THAT STRUCK ME
THE MOST, AND I SUSPECT THIS
WILL COME AS NO SURPRISE AND IT
IS INTERESTING TO ME THAN A
NON-PROFIT ORGANIZATION CAN PLAY
MANY, MANY DIFFERENT ROLES EVEN
IN THE SPACE OF FUNDING EARLIER
MID CAREER INVESTIGATORS AND IT
SEEMS LIKE SOMETIMES YOU ARE
ACTUALLY FUELING THEIR ABILITY
TO CARRY OUT A BUNCH OF STUDIES
AND THEY PERHAPS WANT TO DO TRY
SO YOU GET THEM OFF THE GROUND
AND KEEP THEM ENGAGE IN WHAT IT
IS YOU ARE SOCIETY IS DEDICATING
TO DOING AND I THINK SOMETIMES
ALSO YOU HAVE THE OPPORTUNITY TO
DIRECT WHERE THE FIELD AND
MAKING FUNDING OPPORTUNITIES
AVAILABLE THAT ARE TARGETED IN
SOME WAY OR THAT RESPONDED TO
SOME NEED OF YOUR PATIENT
POPULATION AND FAMILIES OR OF
YOUR ORGANIZATIONS SO IF I'M THE
DIRECTOR OF A NON-PROFIT THAT
HAS JUST MOVED INTO THE
FINANCIAL SITUATIONS WHERE WE
CAN THINK ABOUT FUNDING YOUNG
INVESTIGATORS LET'S SAY WERE IN
THAT SPECTRUM SHOULD I THINK
ABOUT BEING?
DO I PUT OUT SOMETHING VERY
TARGETED THAT I KNOW IS EXACTLY
WHAT OUR PATIENTS NEED IN THE
NEAR TERM OR DO I PUT OUT
SOMETHING THAT WILL HELP ME
INVEST IN THE LONG-TERM FOR THE
CAREER OF SOMEONE WHO IS JUST
GETTING STARTED.
>> I PUT MY HAND UP HERE.
IF I YOU MAY, I THINK ALL OF THE
ABOVE IF WE CONSIDER FINANCE AND
ALL OF THE NON FINANCIAL SUPPORT
THAT YOU CAN DO AND IF I LIKE IT
IT TO ICE CREAM, MANY DIFFERENT
FLAVORS OF ICE CREAM COME FROM
MANY DIFFERENT ORGANIZATIONS AND
ICE CREAM IS STILL DELICIOUS SO
SO ONE OF THE EXAMPLE ARE WE
HAVE A SMALL PERCENTAGE OF OUR
PORT GOAL THAT GETS DIRECTED BUT
I THINK THAT ONE OF THE THINGS
WE DO SO OVER THE LAST COUPLE OF
YEARS WE'VE HAD YOU KNOW JUST TO
MAKE SURE WE HELP TO SUPPORT
EVERYTHING AND WE LET THEM
DECIDE WHICH ARE THE MOST, THE
HIGHEST REACHING ABSTRACT AND
ALSO I WANTED TO ADDRESS WHAT
JENNIFER WAS SAYING ABOUT THE
COVID I THINK THAT A LOT OF OUR
ORGANIZATIONS HAVE BEEN HELPING
RESEARCHERS AND WE'VE GIVEN OUT
WHEN POEM COME HAVE TO DIRE NEED
AND WE'VE ALSO ALLOWED
REBUDGETING SO THERE'S A LOT OF
THINGS THAT WE CAN DO AND THE
ADD VOICE TO THE SCIENTISTS ARE
YOU CAN'T GET IT IF YOU DO ASK
SO KEEP IN CONTACT WITH YOUR
NON-PROFIT GRANT ADMINISTRATOR
AND ANYBODY ELSE WANT TO ANSWER
IN HERE?
I JUST SAID EVERYTHING.
ALL OF THE ABOVE AND ALL FLAVORS
OF ICE CREAM ARE DELICIOUS.
>> ONE THING I WOULD SAY IS IT
WAS SOMETHING THAT WAS MENTIONED
OFF HAND AND ONE OF THE EARLIER
SPEAKERS IN THE DAY AND IT'S
JUST IF YOU START A DISEASE THAT
YOUR KID HAS AND SOMEONE I THINK
JUST MENTIONED A FEW MINUTES
AGO, YOU START WORKING WITH
OTHER PEOPLE ON Facebook AND
RAISING MONEY AND THAT CAN BE
THE THING THAT BRINGS AWARENESS
FOR SCIENTISTS AND RESEARCH AND
I MEAN Dr. MORROW KNOWS THIS
BUT THERE'S A LOT OF MEDICINE
OUT THERE SO LIKE EVEN IF YOU
WANTED TO JUST DO NEUROSCIENCE,
IT'S HARD TO GUARANTEE THAT YOUR
DISEASES OF TAUGHT AND LIKE THE
OVERVIEWS AND OF NEUROSCIENCE
FOR MEDICAL STUDENTS AND
GRADUATE STUDENTS SO WHAT PEOPLE
ACTUALLY END UP DOING AND IN
TERMS OF THE RESEARCH CAREER CAN
BE ALMOST LIKE A RANDOM WALK
LIKE SOME PEOPLE MIGHT JUST END
UP WORKING ON RED SYNDROME
BECAUSE YOU KNOW, OTHER PEOPLE
WERE WORKING ON IT BUT IF YOU
HAVE A RARE DISEASE AND YOU WANT
TO BE FUNDED, IF YOU ARE ABLE TO
SEE START GOING TO CONFERENCES
LIKE AAS BECAUSE THAT IS A TERM
EPILEPSIES IS AND YOU CAN GET
PEOPLE'S ATTENTION AND PEOPLE
ARE WORKING IN IT AND YOU HAVE
THAT MONEY THAT CAN BE AND THERE
WILL BE DOING AT LEAST EARLY
STAGE IF NOT LATER STAGE
RESEARCH ON THE DISEASE AND THEY
FILE AWARE THE MONEY IS SO IF
THAT MEANS THAT THEY END UP
TURNING THEIR GENETIC KNOWLEDGE
TO THIS KIND OF RARE DISEASE AND
AS LONG AS SOMEONE IS GOT LIKE A
PASSIONATE COMMITMENT TO THE
PATIENTS OF THAT DISEASE AND
PEOPLE WILL ACTUALLY START TO DO
THAT RESEARCH.
>> WE HAVE A MINUTE LEFT BUT
THERE'S A QUESTION I WANT TO
POSE THAT PENNY DACKS PUT IN THE
CHAT.
HOW CAN A NON-PROFIT MEASURE
SUCCESS IN ITS INVESTMENTS AND
EARLY CAREER AND IF A FUNDED
TRAINEE LEAVES ACADEMICS
RESEARCH A SUCCESS OR A FAILURE?
>> JENNIFER OR ERIC, DO YOU WANT
TO ANSWER?
>> I CAN GIVE MY THOUGHTS AND
IT'S NOT NEARLY MUFF FOR THIS
LOAD OF OF A QUESTION BUT IT'S
SUCH AN IMPORTANT ONE AND THE
SHORT ANSWER I GIVE IT REALLY
DEPENDS ON YOUR MISSION AND YOUR
GOAL IS AND FOR AN ORGANIZATION
LIKE MINE, OUR GOALS REALLY TO
FURTHER RESEARCH AND TO TREAT
AND CARE EPILEPSIES SO FOR US,
THAT CAN HELP IN A LOT OF
DIFFERENT VENUES, RIGHT, THEY'RE
NON PROFITS DOING THAT AND
THEY'RE INDUSTRY COMPANIES DOING
THAT AND CERTAINLY ACADEMIC
RESEARCH IS IF THAT COULD HAPPEN
IN TERMS OF HOW IT IMPACTS OUR
MISSION.
>> ERIC?
>> I DON'T KNOW THAT I HAVE MORE
TO SAY.
I THINK THAT WAS A GOOD
DISCUSSION OF THAT SO MEASURING
OUTCOMES AND I CAN'T SPEAK TO --
PEOPLE THE PEOPLE FROM THE
NON-PROFIT AND LIKE JENNIFER AND
DIANNE SHOULD SPEAK T TO THAT.
NATURALLY THERE ARE STANDARD
MEASURES AND OF ULTIMATELY TO
DEPENDS ON WHAT THE GOALS ARE
AND TREATMENTS AND GETTING
RAISING AWARENESS AND
INFORMATION AND AS WELL AS
PAPERS AND FUNDING AND IF I CAN
SPEAK TO GENERALLY I DON'T THINK
IT'S A FAILURE IF SOMEONE LEAVES
ACADEMIC RESEARCH BECAUSE THERE
ARE IMPORTANT THINGS TO GO TO
AND MANY IMPORTANT THINGS TO GO
TO SO IT DEPENDS -- OR TO SAD
ROW KA SEE AND POLICY AND WE ARE
SEEING MORE PEOPLE COME BACK AND
HAVE A BLEND EXISTENCE AND SO
WITH THOSE FIELDS SO I THINK IT
SPEAKS TO DO THEY MAINTAIN THE
FIRE AND THE INSPIRATION AND THE
FIGHT TOWARDS THE GOALS AND ONE
OF THE THINGS THAT I WOULD JUST
LIKE TO PICK UP IS THE ISSUE OF
COVID SINCE I DO THINK THAT IS A
SESSION ON TRAINEES AND EARLY
CAREER AND I WAS JUST HAD TWO
CONVERSATIONS THIS MORNING ONE
WITH THE JUNIOR FACULTY TRYING
TO GET HIS FIRST GRANT AND HOW
THE COLLECTION OF THE
PRELIMINARY DATA HAS BEEN
INTERRUPTED REPEATEDLY BY COVID
AND DIFFERENT WAYS MORE ADVANCED
FACULTY MEMBERS WHO SHE'S
THINKING ABOUT PROMOTION AND YOU
KNOW AND HOW IT'S BEEN HARDER TO
MAKE WITH POEM OUTSIDE ARE
INSTITUTE AND VIRTUAL MEETINGS
ARE GREAT AND ZOOM IS HELPFUL IN
MANY WAYS BUT IT'S NOT
CONNECTING IN-PERSON WITH PEOPLE
AND SO MY FINAL COMMENT IS I
LIKED THAT 'EM THINKING HARD
ABOUT HOW WE ALL CAN SUPPORT
THOSE PEOPLE WHOSE CAREERS ARE
MOST VULNERABLE BY THE PANDEMIC.
>> IT CAN ADD WHAT ERIC AND
JENNIFER SAID SO TO ANSWER THE
SECOND QUESTION, SO IT DOESN'T
MATTER FOR US, JUST LOOK AT NON
PROFITS BECAUSE WE ALLOW A P.I.
TO BE FROM ACADEMIA OR
INTRAMURAL GOVERNMENT OK SO TAKE
A LOOK AT INSTITUTIONS AND LOOK
AT THAT.
>> RIGHT.
NIH TAKES EXACTLY THE SAME
VIEWPOINT THAT IN FACT IT IS OUR
JOB TO KEEP SCIENCE HAPPENING
AND TO KEEP EXCELLENCE AS ROBUST
AS IT CAN BE AND WE'VE MADE MANY
NEW MECHANISMS AND VARIATIONS ON
OLD MECHANISMS TO ADAPT TO THE
CIRCUMSTANCES FROM WHICH NONE OF
US CAN HIDE AT THIS POINT.
>> REFER BACK TO PENNY'S
SESSION.
IT'S A PUZZLE.
A JIG SA PUZZLE.
>> MANY.
MANY THANKS BOTH TO THE SPEAKERS
FOR THIS SESSION AND TO OUR
AUDIENCE AND I'M GOING TO TURN
THE THERE WERE OVER TO Dr.
KOROSHETZ.
>> THANK YOU VERY MUCH, NINA AND
THANK YOU SO MUCH ERIC AND
JENNIFER, DAN AND DIANNE.
GREAT SESSION.
I THINK JUST A COUPLE OF
REFLECTIONS FROM THE DAY.
THE ONE THING WE SHOULD ALL
TAKEAWAY IS THERE'S A LOT OF
REALLY GOOD POEM IN THIS SPACE
AND A LOT OF ENERGY IN THIS
VIRTUAL ZOOM ROOM AND I WOULD
SAY THAT WITH REGARD TO THE
TRAINING, DON'T UNDERSTAND
ESTIMATE THE POWER OF THE
CONTAGION OF THIS ENERGY WHEN
PEOPLE ARE LOOKING TO A KA ROAR
CAREERAND MAKING A DIFFERENCE IE
WORLD AND LOOKING HAT A DISEASE.
THERE'S NOT MUCH MORE THAT IS
MORE REWARDING THAN THAT AND SO
THAT DIMENSION IN THE OPENING
REMARKS, THAT COMES FROM THE
DISEASE ORGANIZATIONS AND NOT
USUALLY FROM THE GOVERNMENT SO
YOU HAVE A LOT OF POWER IN
MORPHING THE FUTURE BY
INFLUENCING PEOPLE.
I THINK AFTER THE KIND OF PIE IN
THE SKY, I THINK WE GOT SOME
HEAVY DOSES OF REALITY
THROUGHOUT THE DAY AS PEOPLE
TALK ABOUT THE DIFFERENT
PATHWAYS AND THE NEED FOR
PERSISTENCE AND TO KIND OF
CONVERT THAT ENERGY AND PASSION
TO ACTUAL GETTING PAST THE GOAL
LINE WHICH IS DEVELOPING
TREATMENTS FOR PEOPLE.
LOTS OF DIFFERENT THINGS ARE
NECESSARY AND IT'S ALL ABOUT THE
PATIENTS IN THE END SO THE
DISEASE ORGANIZATIONS THAT ARE
IN TOUCH WITH THE PATIENTS WITH
THE DISEASES HAVE INCREDIBLE
VALUE AND THE PROCESS OF MOVING
TREATMENTS FORWARD, ESPECIALLY
WHEN IT COMES TO CLINICAL
TRIALS, THAT'S REALLY WHERE
THINGS HAPPEN AND I THINK WE'VE
HEARD THAT FROM THE
HYDROCEPHALUS TALK AND THE JONAS
FOUNDATION AND THEIR ABILITY TO
MOVE THEIR ENROLLMENT TO WHERE
IT NEEDED TO BE AND IT'S TRUE
ABOUT SO MANY DIFFERENT
SPECIFICALLY THE MORE LESS
COMMON DISEASES.
WE HEARD A LOT ABOUT THE
FOUNDATION FOR NIH WHICH IS SET
UP BY CONGRESS TO DEVELOP THESE
KIND OF PARTNERSHIPS.
THEY KEEP THE GOVERNMENT PEOPLE
IN THEIR SWIM LANES SO THEY
DON'T END UP IN JAIL AND THINK
MANAGEMENT AND DEVELOPING
PARTNERSHIPS WITHIN THE LEGAL
FRAMEWORK THAT THE GOVERNMENT
FOLKS CAN WORK IN AND WE HEARD
ABOUT THE BUSINESS OF DEVELOPING
TREATMENTS AND THE PROGRAMS MA
WE HAVE HERE IN NIH WHICH FUND
WHETHER THEY'RE TRYING TO
DEVELOP TREATMENT AND
PARTICULARLY THE SMALL
BUSINESSES ARE OFTEN TIMES
THEY'RE SWIM LANE IS THE RARE
DISEASES AND THERE'S REAL
IMPORTANT LEGAL AND MANAGEMENT
FRAMEWORKS THAT YOU HEARD ABOUT
TODAY AND EXAMPLES WHERE THEY
HAVE HAD SUCCESS IN GAINING
FUNDS FROM INDUSTRY OR FROM NIH
AS WE HEARD FROM THE CASE.
WE TALKED ABOUT THE ACADEMIC
PARTNERS WHERE PEOPLE ARE IN
TRAINING AND YOU CAN PUT YOUR
HOOKS INTO PEOPLE AND GET THEM
INTERESTED IN YOUR DISEASE AND
WE HAVE INVESTIGATORS WHO HAVE
DECIDED TO MAKE THEIR CAREER IN
UNDERSTANDING SO DEVELOPING
RELATIONSHIPS WITH THOSE PEOPLE
IS BENEFICIAL TO BOTH SIDES THEY
GET A LOT OF FULFILLMENT OUT OF
BEING PART OF THIS BIGGER
COMMUNITY AND THEY'RE WORKING IN
THEIR LABS DAY AND NIGHT AND A
LITTLE BIT OF AN ISOLATION AND
WHEN THEY COME TO A MEETING BY
DISEASE ORGANIZATION, LISTEN TO
THE PATIENTS AND LISTEN TO THEIR
CAREGIVERS AND LISTEN TO THE
OTHER INVESTIGATORS WHO ARE ALL
ENGAGE AND I THINK THAT'S A VERY
FULFILLING PART OF THEIR CAREER
AND THEY'RE THE PEOPLE WHO ARE
GOING TO HAVE THIS DISCOVERIES
THAT MAKE THINGS HAPPEN.
AND THE FUTURE IS REALLY IN THE
NEW PEOPLE WHO ARE COMING INTO
THE FIELD OF THE POOLS THAT ARE
COMING OUT AS I MENTIONED IN MY
TALK AND I THINK THEY'LL BLOW
AWAY WHAT WE HAVE NOW AND SO THE
ABILITY TO INFLUENCE AND GET
YOUNG PEOPLE TRAINED IN
TRANSLATION WHETHER IT'S THROUGH
A BOOTCAMP OR WHETHER IT'S
DISEASE MEETINGS AND I THINK
IT'S SO IMPORTANT IN GENERATING
THE ARMY THAT IS GROUND ZERO TO
SOLVE THE PROBLEMS THAT WE'VE
BEEN ABLE TO PUT OUT ON THE
TABLE AND SO DEVELOPING THE
WELCOME COMMUNITY FOR YOUNG
PHYSICIANS FOR YOUNG PHD
SCIENTISTS AND FOR ALL THE OTHER
PROFESSIONALS AND IS REALLY WHAT
IS A BIG INVESTMENT AND OFTEN
TIMES IT'S NOT SO EXPENSIVE.
A LITTLE MORE EXPENSIVE HAN WHEN
I WAS YOUNG BUT IT'S STILL A
FAIRLY INEXPENSIVE WAY TO REALLY
BUILD YOUR KADRI OF
INVESTIGATORS MOVING FORWARD SO
I WAS REALLY IMPRESSED.
EVERY YAR WE THINK OH BOY,
NON-PROFIT FORUM CAN'T BE BETTER
BUT IF SEEMS LIKE ONCE AGAIN YOU
HAVE EXCELLED AND IT GETS BETTER
AND BETTER AND IT'S BETTER THIS
YEAR THAN EVER AND SO THANK YOU
TO ALL THE PEOPLE WHO PUT THEIR
WORK IN.
TOMORROW WE'LL HEAR ABOUT
SUCCESS STORIES AND DIFFERENT
ORGANIZATIONS HAVE HAD AND WE
LIKE TO HAVE THESE SHOWERED AND
ALSO WE'LL HAVE TALKS AND
DISCUSSIONS ABOUT HOW WE MOVE TO
BE MORE INCLUSIVE IN THE
RESEARCH THAT WE DO AND THE
PATIENTS THAT WE SEARCH AND THEN
THERE'S A SESSION AS ME SAID
TALK AMONGST YOURSELVES TO LEARN
FROM EACH OTHER AND THESE BREAK
OUTS AND SO THERE'S GOING TO BE
IN THE LINK IN THE CHATBOX OF
PEOPLE TO TAKE THIS SURVEY THAT
WILL INFORM THE BREAK OUT
SESSION AND ASSIGNMENTS TOMORROW
SO IF YOU HAVEN'T DONE THAT,
PLEASE DO THAT NOW AND IT WILL
HELP US TOMORROW AND THERE'S
OPPORTUNITIES STILL AVAILABLE TO
TALK TO THE PROGRAM DIRECTORS
AND EVERY SINGLE DISEASE HAS A
PROGRAM DIRECTOR IN CHARGE OF
THAT DISEASE AND SOME PROGRAM
DIRECTORS HAVE ONE DISEASE LIKE
THE STROKE FOLKS AND SOME LIKE
JOE MORRIS PROBABLY HAVE 30
DIFFERENT DISEASES IN THEIR
PORTFOLIO AND MOSTLY RARE
DISEASES AND THE PROGRAM
DIRECTORS ARE HERE TO HELP YOU
ACCESS THE SN NIH RESEARCHERS OS
IN YOUR FIELD BECAUSE IT'S
REALLY DOVE TAILS WITH OUR
MISSION AND ALMOST ALL THE CASES
THAT YOU HAVE.
THANK YOU AGAIN TO THE GROUP AND
LOOK FORWARD TO DISCUSSIONS
TOMORROW.
HAVE A GOOD EVENING.
I WISH WE WERE IN-PERSON.