>>WELCOME TO
DAY 2 OF NON-PROFIT FORUM.
ON DAY ONE WE STUCK WITH OUR
THEME WHICH WAS HOW TO WORK WITH
AND DOT DOT DOT WHICH MEANT
WHEREVER YOUR PARTNER'S GOING TO
BE WHETHER IT'S INDUSTRY OR
ACADEMIA OR GOVERNMENT.
WE HEARD HIGH LEVEL
PRESENTATIONS AND GETS IN THE
ACTIONABLE SPACE THE
ORGANIZATIONS ASSEMBLED TO BE
MORE EFFECTIVE IN GARNERING THE
PARTNERS THAT THEY NEED TO
ADVANCE THEIR PARTICULAR
MISSIONS.
TODAY IS MORE OF INTERNAL WALK
DOWN OURSELVES AT THE GOVERNMENT
AND NONPROFIT LEVEL.
A MAJOR FEATURE OF THE
NON-PROFIT FORUM IS FOR THE
DISEASE ORGANIZATIONS TO SHARE
THEIR LESSONS LEARNED TO NETWORK
AMONG THEMSELVES BECAUSE IN MANY
ASPECTS THE THINGS THAT YOU'RE
TRYING TO DO THE PEOPLE WHO HAVE
TRIED IT BEFORE ARE THE BEST
ONES TO GIVE YOU ADVICE.
AND THEN ALSO BRAIN STORMING ON
HOW TO SOLVE SOME OF THE ISSUES
THAT FACE THE DISEASE
ORGANIZATIONS I THINK IS
IMPORTANT FUNCTION OF THE
NON-PROFIT FORUM TO SERVE AS THE
FOUNDATION FOR THAT DISCUSSION
TO HAPPEN.
WE'RE GOING TO START OFF WITH
OUR USUAL FEATURE CALLED SUCCESS
STORIES AND THESE ARE CLEARLY
EVERYONE IS WORKING TOWARDS THE
GOAL OF TREATMENT AND THERE'S
UPS AND DOWNS ON THAT ROAD.
WE LIKE TO TALK ABOUT SOME OF
THE UPS THAT HAVE OCCURRED IN
THE GROUP AND WE SEARCH FOR
THOSE AND PICK OUT A COUPLE OF
WHAT WE CALL SUCCESS STORIES AND
TISH HEVEL WILL MODERATE AND
THEIR PROGRAM IS A SUCCESS STORY
AND TISH HAS BEEN UNBELIEVABLY
EFFECTIVE AT DEVELOPING A
PROGRAM TO ALLOW PEOPLE TO
DONATE THEIR BRAINS FOR RESEARCH
WHICH IS SO IMPORTANT NOW TO
EXAMINE BRAIN AT THE SINGLE CELL
HEAVILY IN DISEASES AND CONTROL
GROUPS.
TISH WAS RECENTLY ON NPR AND
THAT SPURRED A LOT OF INTEREST
IN THIS TOPIC.
HATS OFF TO TISH AND TOE THE
OTHER FOLKS IN THAT SESSION
TALKING ABOUT THE THINGS THEY
WERE ABLE TO PULL OFF IN THE
RECENT TIME FRAME.
NOW WE'LL HAVE A DISCUSSION HOW
TO ACHIEVE EQUITY AND
INCLUSIVITY IN THE WORK WE DO.
I THINK OFTEN TIMES WE'RE VERY
FOCUSSED ON THE GOAL IN FRONT OF
US AND THE PEOPLE WHO KIND OF
COME TO THE FRONT LINES BUT
THERE'S A LOT OF PEOPLE LEFT
BEHIND WHO DON'T KNOW WE'RE
DOING AND MIGHT BE INTERESTED IF
THEY KNEW WHAT WE WERE DOING OR
MAY NEED SOME SPECIAL ATTENTION
TO ACTUALLY BRING THEM IN THE
FOLD PARTICULARLY PEOPLE WITH
LOW SOCIO ECONOMIC STATUS WHO
DON'T HAVE THE WHERE WITH ALL
TO GET IN THE MIX AND HOW TO DO
THAT IN THE NON-PROFIT SPACE.
IN THE AFTERNOON THERE'LL BE
BREAKOUT SESSIONS ACCORDING TO
TOPICS YOU HOPEFULLY SIGNED UP
FOR IN THEIR SURVEY AND WE'LL
COME BACK AT THE END TO SHARE
WHAT CAME OUT OF THE BREAKOUTS
AND SUM UP THINGS FROM THE DAY.
BUSY DAY AHEAD OF US.
HIGH QUALITY SPEAKERS AND I
THINK CERTAINLY THE PASSION AND
ENERGY FROM YESTERDAY IS CLEARLY
GOING TO BLEED INTO TODAY AND
KEEP US GOING.
WITH THAT LET ME TURN IT OVER TO
OUR FIRST SESSION AND OUR
MODERATOR TISH HEVEL FROM THE
BRAIN DONOR PROJECT.
>> THANK YOU, DR. K. AND THANK
YOU FOR THE KIND WORDS.
I APPRECIATE THAT.
AS YOU MENTIONED, MOST OF US ARE
IN THE BUSINESS OF FINDING CURES
FOR NEUROLOGIC DISORDERS OR
SUPPORTING PEOPLE WHO HAVE THOSE
DISEASES OR BOTH.
WHEN WE TALK ABOUT SUCCESS
STORIES WE'RE TALKING ABOUT THE
WINS ALONG THE WAY.
NOT THE ULTIMATE GOAL YET BUT
THE THINGS HELPING US GET
CLOSER.
DR. KOROSHETZ MENTIONED I'M WITH
THE BRAIN DONOR PROJECT AND
HAPPEN TO REPRESENT AN
ORGANIZATION THAT HELPS PEOPLE
BECOME BRAIN DONORS WHEN THEY
DIE.
SO WE MEASURE THE WINS IN TERMS
OF FOLKS WHO TAKE STEPS NOW TO
PLAN TO BE BRAIN DONORS WHEN THE
TIME COMES OR THE SUCCESSES THAT
COME FROM MORE PEOPLE KNOWING
ABOUT THE CRITICAL NEED FOR
BRAIN DONATION AND I'LL TALK
MORE ABOUT THAT A LITTLE BIT
LATER IF WE HAVE TIME.
BUT THE CONTRIBUTORS TO THIS
PANEL ON SUCCESS STORIES HAVE
CRITICAL, CRITICAL TANGIBLE
ACHIEVEMENTS TO CELEBRATE.
THERE'S STRONG STEPS IN A
SOLIDLY COLLECTION THAT HELP
IMPROVE SCIENCE AND SOME ARE IN
MILESTONES OF DIVERSITY, EQUITY
AND INCLUSION AND WORKING
TOWARDS ENSURING THE SCIENCES
REPRESENTATIVE OF MORE KINDS OF
PEOPLE.
WE KNOW IT'S NOT COMPLETE
OTHERWISE.
WITH US ARE THREE SCIENTISTS WHO
MADE SOME SIGNIFICANT PROGRESS
IN THAT ARENA AND OTHERS.
THEY ARE JIM BECK OF THE
PARKINSON'S FOUNDATION.
AGUSTIN IBANEZ OF THE REDLAT
CONSORTIUM AND KEVIN O'CONNOR
FROM YALE TALKING ABOUT THE
MYASTHENIA GRAVIS FOUNDATION OF
AMERICA.
WE'LL BEGIN WITH DR. BECK.
>> THANK YOU.
I APPRECIATE THE OPPORTUNITY TO
BE INVITED TO THE SESSION AND
TELL YOU A LITTLE BIT ABOUT OUR
SUCCESS.
I'LL TALK ABOUT THE PARKINSON'S
FOUNDATION.
THE GOAL IS TO IMPROVE THE LIVES
OF THOSE LIVING WITH PARKINSON'S
DISEASE THROUGH IMPROVED CARE
AND ADVANCING RESEARCH TOWARDS A
CURE AND LOOK TO EMPOWER AND
EDUCATE OUR GLOBAL COMMUNITY.
WHAT I'LL TALK ABOUT WILL TACKLE
THE GOALS OF OUR MISSION AS AN
ORGANIZATION AND I THINK IS
REPRESENTATIVE OF WHERE I HOPE
RESEARCH CAN TAKE US MOVING
FORWARD PARTICULARLY THROUGH OUR
SUCCESS AND COLLABORATION.
SO PD GENERATION IS A LARGE
AT-HOME STUDY PRIMARILY BECAUSE
OF COVID BUT CAN BE DONE IN THE
CLINIC IN ESSENCE REACHING
15,000 PEOPLE WITH PARKINSON'S
DISEASE BY OFFERING FREE NO-COST
GENETIC TESTING AND COUNSELLING
TO PEOPLE WHO LIVE WITH THE
DISEASE.
THE FOCUS IS RECOGNIZING EVERY
PERSON'S JOURNEY WITH
PARKINSON'S DISEASE IS UNIQUE.
IT'S A DEGENERATIVE NEUROLOGIC
DISEASE WHICH TYPICALLY AFFECTS
PEOPLE IN THEIR 60s OR OLDER AND
UNFORTUNATELY THERE'S NO CURE.
WE SEE EVERYONE'S PROGRESS AS
PART OF THAT DISEASE IS UNIQUE
AND GENETICS MAY SERVE AS A KEY
TO UNDERSTANDING NOT ONLY THE
BIOLOGY BUT HELP DIAGNOSE AND
TREAT AND FOCUSSING ON THE
TREATMENT WITH GENETICS IS
UNIQUE IN THE REALM OF
NEUROLOGIC DISEASE AND HAVE SEEN
IT HAPPEN IN THE REALM OF CANCER
WHERE THE IDEA OF PRECISION
MEDICINE IS THERE.
HERE WE TALK ABOUT PEOPLE WHO
MAY HAVE A CANCER DIAGNOSIS AND
IT HAS A UNIQUE MUTATION WHERE A
SPECIFIC DRUG MAY TARGET THAT
MUTATION AND STOPS THE GROWTH OF
TUMOR CELLS.
I THINK WE'RE ON THE CUSP OF
THAT HAPPENING WITH PARKINSON'S
DISEASE.
IT'S YET TO BE PROVEN BUT THAT'S
THE DIRECTION OF THE FUTURE OF
PARKINSON'S THERAPIES.
THERE'S A NUMBER OF
PHARMACEUTICAL COMPANIES WHO
LOOK TO TAKE THIS LEAP AND
TREATMENT WITH PEOPLE WITH
PARKINSON'S DISEASE WITH EARLY
STAGE RESEARCH.
THERE'S A PROBLEM THE COMMUNITY
IS NOT READY AND THE GOALS OF PD
GENERATION ARE SIMPLE.
THROUGH GENETIC TESTING AND
PEOPLE WITH PARKINSON'S WE HOPE
TO ACCELERATE PRECISION MEDICINE
FOR CLINICAL TRIAL FOR PD AND
WANT TO UNLOCK THE IMPROVED CARE
AND EMPOWER PATIENTS TO TAKE
CONTROL OF THEIR DISEASE.
THE FACT IS GENETIC TESTING IS
NOT ROUTINE CARE FOR PEOPLE WITH
PARKINSON'S DISEASE AND AS
COMPANIES LOOK TO RECRUIT PEOPLE
NO ONE KNOWS WHO HAS A GENETIC
FORM.
IT'S RELATIVELY RARE.
10% TO 15% HAVE A GENETIC FORM
OF PD AND OUR EFFORTS ARE TO
IDENTIFY THOSE INDIVIDUALS WHO
MAY HAVE GENETIC PD AND CAN
PARTICIPATE IN THE CLINICAL
TRIAL TO ACCELERATE THEM SO THEY
DON'T FAIL FOR LACK OF
RECRUITMENT.
JUST NUTS AND BOLTS IS IT'S
ELIGIBLE FOR EVERY ADULT WHO HAS
A DIAGNOSIS OF PARKINSON'S
DISEASE AND WE'RE OFFERS FREE
GENETIC TESTING.
IT'S AN EXPENSIVE CLINICAL GRADE
TEST AND NOT ONLY USE IT FOR
RESEARCH PURPOSES BUT RETURN
RESULTS WITH CONFIDENCE THAT
WHAT WE'RE REPORTING BACK IS
ACCURATE.
WE OFFER GENETIC COUNSELLING IN
ENGLISH AND SPANISH.
THAT'S UNIQUE THESE DAYS AND YOU
MAY HEAR GENETIC TESTING
OFFERING DOZENS OF GENES.
WE FOCUS ON SEVEN SPECIFIC GENES
RELEVANT TO PARKINSON'S DISEASE
WE THINK THE MOST COMMON OF WHAT
SAY RARE FORM OF PARKINSON'S
DISEASE AND THE MOST RELEVANT
LOOKING AT THE ROAD MAP OF WHERE
PHARMACEUTICAL COMPANIES ARE
LOOKING TO ENGAGE WITH POTENTIAL
EXPERIMENTAL MEDICINES.
WE BANK DNA FOR FUTURE USE AND
WE LOOK TO HAVE ABOUT 100 SITES
IN THE U.S. PARTICIPATING IN OUR
STUDY.
THE RESULT HAS BEEN TREMENDOUS.
WE HAVE NEARLY 5,000
PARTICIPANTS IN A LITTLE MORE
THAN TWO YEARS FROM ALL 50
STATES PLUS PUERTO RICO AND THE
DEMAND HAS BEEN TREMENDOUS.
PEOPLE ARE EXCITED BY THIS.
I HAVE TO EMPHASIZE OUR SUCCESS
IS NOT JUST SOMETHING DRIVEN BY
THE FOUNDATION ALONE.
IT'S BEEN THROUGH CLOSE
COORDINATION WITH OUR COMMUNITY
AND PARTICULARLY THE COMMUNITY
WHO ARE NOT TYPICALLY
REPRESENTED IN RESEARCH PROCESS.
WE ARE A PARKINSON'S FOUNDATION
AND WE HAVE PEOPLE WITH
PARKINSON'S FIRST WE DISCUSSED
IT WITH OUR ADVISORY COUNCIL A
BOARD LEVEL WITH PEOPLE WITH
PARKINSON'S AND CARETAKERS AND
PARTNERED WITH PEOPLE WITH
PARKINSON'S TO HELP INFORM OUR
STUDY DESIGN AND CONSENT PROCESS
AND ONCE ESTABLISHED THE STUDY
AS WE TYPICALLY DO, WE INCLUDED
A PERSON WITH PARKINSON'S AS A
FULL MEMBER OF THE STEERING
COMMITTEE AND THIS SAY PERSON
WITH PARKINSON'S WHOSE VOICE IS
HEARD AND HAS PROVIDED ESSENTIAL
FEEDBACK AS WE LOOKED TO MOVE
FORWARD.
WE'VE ALSO LOOKED TO TRY TO
REACH OUT TO COMMUNITIES NOT
TYPICALLY REPRESENTS IN
RESEARCH.
WE MADE CERTAIN OUR STUDY COULD
BE CONDUCTED COMPLETELY IN
SPANISH FROM OUR MATERIALS
WRITTEN IN SPANISH TO PHYSICIANS
ABLE TO RECRUIT IN SPANISH TO
COUNSELLING DONE IN SPANISH
COMPLETELY.
FOR THOSE INDIVIDUALS WHO FIND
SPANISH IS THE LANGUAGE THEY
WANT TO SPEAK IN PARTICULARLY
AROUND MEDICAL ISSUES, IT'S BEEN
INCREDIBLY HELPFUL.
WE ALSO LOOKED TO TRAIN
PROFESSIONALS THAT ACT AS A
LIAISON BETWEEN THE HISPANIC
COMMUNITY AND THE CLINICAL
REALM, THE PHYSICIANS.
BY TEACHING THEM ABOUT
PARTICIPATION IN RESEARCH AS
WELL AS GENETICS, WITH AN
ADVISORY GROUP THEY INCREASED
ENROLLMENT.
THE ENROLLMENT IS NOW CLOSE TO
14% AND WE'VE WORKED HARD TO
REACH OUT TO HISPANIC
COMMUNITIES WITH THE CONTINENTAL
SUCCESS.
AND WE HAD A REPRESENTATION
ABOUT PARKINSON'S DISEASE AND
MOREHOUSE AND HAVE MET WITH
SUCCESS ON RECRUITING THOSE WHO
ARE BLACK AND AFRICAN AMERICAN
ANCESTRY WITHIN THE PD
GENERATION STUDY.
WE'VE ALSO WORKED WITH AMERICAN
INDIAN AND ALASKAN NATIVE
COMMUNITIES AND THESE
COMMUNITIES THROUGH PARTNERSHIPS
HAVE BEEN ABLE TO REACH
COMMUNITIES NOT WELL REPRESENTED
WITHIN RESEARCH AND PARTICULARLY
GENETICS RESEARCH AS A WHOLE.
HAVING REPRESENTATION IS
IMPORTANT SCIENTIFICALLY AS WELL
BECAUSE GENETICS ARE SO
IMPORTANT TO UNDERSTAND A
DISEASE THAT WHERE WE HAVE SUCH
LITTLE INFORMATION ABOUT WHY
PEOPLE DEVELOP THE DISEASE OR
HOW IT PROGRESSES OR HOW TO STOP
IT.
WE'VE ALSO WORKED WITH LARGE
PARTNERS AS WELL IT'S IMPORTANT
TO ACKNOWLEDGE FROM PHARMA LIKE
BIO GEN AND AND WE WORK WITH
OTHER NONPROFITS LIKE THE FOX
FOUNDATION AND GP2 WHICH IS
INTERESTED IN THE RESEARCH
COMPONENT OF THE GENETIC
COMPONENT OF THE TESTING AND A
LARGE INTERNATIONAL SOCIETY.
I WANT TO CONCLUDE BY SAYING IF
PEOPLE ARE INTERESTED IN WANTING
TO LEARN MORE FEEL FREE TO YOUR
HOLD PHONE UP TO THE SCREEN AND
IT THE TAKE YOU TO OUR WEBSITE
AND YOU CAN LEARN MORE ABOUT THE
STUDY AND CAN E-MAIL US AT
GENETICS AT PARKINSON'S.org AND
MY E-MAIL SHOULD BE AVAILABLE
LATER AND HAPPY TO TAKE
QUESTIONS OUTSIDE THE PANEL.
THANK YOU, TISH.
>> THANK YOU.
GREAT STUFF.
IT'S EXCITING.
THE NEXT PERSON IN THE LINE UP
IS DR. AGUSTIN IBANEZ.
IF YOU'RE READY TO GO I CANNOT
DO IT BY MYSELF.
SO I FIRST OF ALL I WANT TO TELL
YOU SOME ACTIONS WE'VE BEEN
DEVELOPING IN LATIN AMERICA.
FIRST, QUESTION IS WHY LATIN
AMERICA.
AS YOU KNOW LATINOS IN U.S. AND
ARE UNDER REPRESENTED IN
CLINICAL TRIALS AND RESEARCH BUT
ESPECIALLY IN LATIN AMERICA.
PATIENTS AND FAMILIES WITH
DEMENTIA ARE FACING HUGE
BARRIERS AND FRAGMENTED UNSTABLE
HEALTH SYSTEM AND THERE'S AMONG
100% TO 250% INCREASE IN THE
FUTURE AND THERE'S SOCIAL
DETERMINATES AND THIS IS A
DIVERSE UNDER REPRESENTED
POPULATION AND FACING AN
ENORMOUS AMOUNT OF CHALLENGES.
CAREGIVERS IN LATIN AMERICA ARE
AROUND THOSE WHO DON'T HAVE
ACCESS TO FORMAL LONG-TERM CARE
AND DON'T HAVE TRAINING OR
PREPARATION.
THERE'S A HUGE GENDER BIAS.
MOST CAREGIVERS ARE WOMEN,
MOTHERS, CAREGIVERS AND WIVES.
SO THE FIRST THING WE TRIED TO
DO IS TO TRY TO THINK TOGETHER
LIKE SEVEN YEARS AGO WE WERE
ABOUT CLINICIANS OR RESEARCHERS
WE REALIZED WE ARE FACING WE
DON'T HAVE A CHANCE TO GET THE
DIAGNOSIS FOR PATIENTS
ESPECIALLY THOSE WITH LOW
ECONOMIC SETTINGS AND THE
RESEARCH FOUND IN LOCAL
COUNTRIES ARE REALLY SMALL AND
NOT ENOUGH TO DEVELOP A STRONG
RESEARCH.
THE GROUPS WORK IN ISOLATION AND
THEY'VE COORDINATED AND WE WERE
ABLE TO INTEGRATE THE DIFFERENT
COUNTRIES AND NOW WE'RE MORE
THAN 250 EXPERTS IN THE REGION
AND GROUPS AND STRONG CLINICAL
GROUPS.
WE WANT TO TRANSFORM THE KIND OF
CHALLENGES INTO OPPORTUNITIES.
THE FIRST STEP WE WERE ABLE TO
PERFORM WAS TO EXPAND THE
RESEARCH IN LATIN AMERICA
FOUNDED BY THE NIH AND
ALZHEIMER'S ASSOCIATION AND
SEVERAL PRIVATE INVESTMENT WE
WANT TO IDENTIFY WHAT MAKES
LATIN AMERICANS SPECIAL.
THERE'S TWO SOURCES OF RISK
FACTORS ONE IS THE SOCIAL
DETERMINATES OF HEALTH AND
THERE'S HIGH INEQUALITY AROUND
THE WORLD AND THERE'S VIOLENCE,
INSECURITY, HOUSING, ECONOMIC
STABILITY THAT IMPACTS THESE AND
THERE'S MODIFIABLE RISK FACTORS
LATIN AMERICA HAVE 56% OF THE
PRESENTATION OF DEMENTIAS BY
RISK FACTORS AND THERE'S
DIFFERENT ANCESTRIES AND THE
OTHER AREAS ARE EXTREMELY UNDER
REPRESENTED.
IN REDLAT WE'RE NOW COLLECTING
PARTICIPANTS AND DATA AND WE
HAVE COLLECTED SOME AND WE'RE
GOING TO COMPARE THE GENETIC
STUDIES IN THE LATIN AMERICA
POPULATION AND WE ARE ALSO
DEVELOPING FOR NEUROIMAGING AND
COGNITIVE TASKS HAVE BEEN FOR
EUROPEAN OR U.S. POPULATIONS.
SO WE HAVE BEEN DOING A HUGE
WORK IN TRYING TO HARMONIZE THE
PROTOCOL ACROSS THE CONSORTIA
AND WE ARE COMPARING THIS
AGAINST U.S. PARTICIPANTS.
SO WE CREATED THE INSTITUTE THAT
IS A LOCAL WILL LOCATION IN
CHILE TRYING TO DEVELOP A LOCAL
TEAM AND THIS IS AN INSTITUTE AT
UCSF AND WE PROVIDE 18 FACULTY
POSITIONS, 100% DEVOTED TO
RESEARCH AND WE ARE OFFERING
GRANTS FOR LATIN AMERICAN TEAMS
AND A MULTI-OMICS APPROACH TO
UNDERSTAND DEMENTIA.
SO IN THE LESS THAN SIX YEARS
WE'VE BEEN ABLE TO CREATE A
POWERFUL TEAM.
WE HAVE PUBLISHED HUNDREDS OF
PAPERS AND WELL RENOWN JOURNALS
AND INVOLVED THE COMMUNITIES AND
WE HAVE MANY WORKING IN MANY
ASSOCIATIONS AND HAVE 12 PILOT
GRANTS TO INCREASE THE SIGNS OF
DEMENTIA AND WORKING ON STUDIES
AND WE'RE ALSO CREATING A WAY TO
LOOK AT DIVERSE POPULATIONS
INCLUDING POLICY MAKERS AND
PATIENTS, FAMILIES AND OTHER
PROFESSIONS.
WE HAVE A PLAN FOR DEMENTIA
INVOLVING EPIDEMIOLOGICAL
STUDIES AND CLINICAL TRIALS AND
RESEARCH INITIATES AND PUBLIC
POLICIES.
WE'RE GOING TO CREATE THE FIRST
FELLOWSHIP TO EMPOWER THOSE IN
THE REGION WHO WANT TO MAKE AN
IMPACT IN RESEARCH OR FOR THE
FAMILIES OF PATIENTS WITH
DEMENTIA.
WE ARE ALSO STARTING TO CREATE A
KIND OF EXTENDED MENTORING AND
TEACHING ACROSS DIFFERENT
OPPORTUNITIES IN THE REGION.
THE NEXT YEAR WE WILL START TO
INCREASE THE Ph.D. PROGRAM ON
HEALTH IN THE REGION.
WE ARE SUPPORTING MANY CENTERS
IN THE REGION THROUGH FACULTY
THROUGH CHANGE AND TRAINING AND
WE ARE TRYING TO DEVELOP AN
INTERNATIONAL SUPPORT BY
DIFFERENT EUROPEAN AND
U.S. INSTITUTIONS.
SO WE'RE TRYING TO SUPPORT THE
CAPACITY BUILDING.
WE HAVE BEEN MAKING GOALS FOR
THE GROUP FOR PEOPLE WITH
DEMENTIA AND IMPAIRMENT IN THE
REGION.
THE NEED TO DEVELOP A NOVEL
APPROACH LIKE BRINGING THE
INVESTMENT FOR THE FUTURE OF
BRAIN CAPITAL IN THE REGION AND
TRYING TO CHANGE THE PRACTICES
TO IMPROVE THE SUPPORT FOR THE
CAREGIVERS IN THE REGION.
WE HAVE PROVIDED MANUALS IN
NATIVE LANGUAGE TRYING TO
HARMONIZE THE RESEARCH AND
TRYING TO INTRODUCE TELEMEDICINE
AND TOOLS TO IMPROVE THE
CONDITION OF THE PATIENTS.
WE WANT TO TRY TO DEVELOP A
TRULY INTEGRATED APPROACH.
SO WE TRIED TO DO SCIENCE BUT
THE TRANSLATION IS PART OF THE
COMMITMENT AND CAPACITY BUILDING
AND LOCAL IDEAS IN GLOBAL
LANDSCAPE AND WE WANT TO ARE
LOOK AT THE GLOBAL LANDSCAPE AND
INFORM THE CHALLENGES INTO
OPPORTUNITIES.
WE WANT TO DEVELOP THE NETWORK
BUT WE CAN BIG CHALLENGES AHEAD
WE NEED TO GO AND WORK HARDER.
THANK YOU SO MUCH.
I THINK I AM FINISHED NOW.
>> WOW.
THANK YOU.
THAT'S COMPLICATED WORK WITH A
LOT OF PEOPLE.
THAT'S FABULOUS.
OUR NEXT PRESENTER IS DR
DR. O'CONNOR.
>> THANK YOU, TISH.
THANK YOU EVERYBODY FOR YOUR
ATTENTION THIS MORNING.
I'D LIKE TO TURN TO SOME OF THE
WORK WE'VE DONE WITH THE
MYASTHENIA GRAVIS FOUNDATION
AMERICA AND SOME PROGRESS MADE
IN THE LAST FEW YEARS.
SO WE SHOULD BE ON THE SLIDE
THAT SAYS WHAT IS MYASTHENIA
GRAVIS, RIGHT?
>> YES.
I WANTED TO START BY EXPLAINING
WHAT MYASTHENIA GRAVIS IS AND
IT'S SUPPORT THE NINDS.
IT'S NOT A DISEASE CONSIDERED TO
BE STRICTLY NEUROLOGICAL.
IT'S NEUROMUSCULAR AND IT'S A
CHRONIC AUTOIMMUNE DISEASE THAT
INTERRUPTS NEUROMUSCULAR
CONDITION.
IT'S RARE.
14 TO 40 OF 100,000 PEOPLE AND
THIS IS WHERE THE NERVE ATTACHES
TO THE MUSCLE AND THE IMMUNE
SYSTEM MAKES ANTIBODIES THAT
INTERRUPT THE TRANSMISSION FROM
THE NERVE TO THE MUSCLE AND
PATIENTS DUE TO THAT BECOME VERY
FATIGUED.
THEY HAVE SEVERE MUSCLE WEAKNESS
AND THEY HAVE IMPAIRED MUSCLE
CONTRACTIONS.
THE DISEASE CAN BE QUITE SEVERE
JUST BY SAYING THERE'S FATIGUE
DOESN'T MEAN TO MINIMIZE IT
COULD BE LIFE THREATENING AND
COULD BE QUITE SERIOUS
INFLICTION.
SO THE MYASTHENIA GRAVIS
FOUNDATION IS THE LARGEST
LEADING PATIENT ADVOCACY
FOUNDATION SOLELY DEDICATED TO
FINDING A CURE IN IMPROVED
TREATMENT FOR MYASTHENIA GRAVIS.
WHAT WE'VE BEEN WORKING ON IS
TRYING TO FIND THE MOST
PROMISING CRITICAL RESEARCH
DISCOVERIES AND TO PROVIDE
PATIENT-CENTERED PROGRAMS AND
MATERIALS TO CONNECT AND ASSIST
MEMBERS OF THE GLOBAL MG
COMMUNITY.
SO MY ROLE I HAVE TWO ROLES I
WEAR TWO HATS.
ONE WITH THE MGFA AND THE OTHER
UNIVERSITY.
I'M THE CHIEF SCIENTIFIC OFFICER
AND PARTNERED WITH THE CHIEF
MEDICAL OFFICER.
THIS IS AN IMPORTANT
DISTINCTION.
I HAVE A Ph.D. AND BENCH
SCIENTIST AND MY PARTNER WHO IS
A CLINICIAN AT YALE IS A
NEUROMUSCULAR SPECIALISTS WHO
TREATS PATIENTS WITH MYASTHENIA
GRAVIS.
WE MAKE A COMPARABLE PAIR FOR
DIRECTING THE SCIENCE AND
MEDICINE OF THE MGFA.
MY ROLE IN RECENT TIMES WITH THE
MGFA HAS BEEN TO GUIDE RESEARCH
AND FUNDING PROGRAMS.
I'LL TALK ABOUT IN THE NEXT
COUPLE SLIDES.
THE MOST RECENTLY WE ORGANIZED
OUR 14th INTERNATIONAL MEETING.
THEY'RE HELD EVERY FIVE YEARS
FOR THE MG COMMUNITY THAT BRING
RESEARCHERS AND CLINICIANS
AROUND THE WORLD TO DISCUSS THE
LATEST FINDINGS.
WE'VE GROWN TO THE POINT NOW
WHERE THAT MEETING WILL BE EVERY
THREE YEARS AND NEXT TIME IT
WILL BE AT AN INTERNATIONAL
LOCATION.
IT'S BEEN HELD IN THE U.S. SINCE
AND WE'RE MOVING TO EUROPE NEXT
TIME IN TWO YEARS COMING.
AT YALE I'M THE PRINCIPAL
INVESTIGATOR AND MY LAP STUDIES
THE MECHANISMS BY D CELLS THAT
MEDIATE THE PATHOLOGY OF
MYASTHENIA GRAVIS.
SO SOME OF THE DIFFERENTIATORS
OF THE MGFA I WANTED TO
HIGHLIGHT WITH RESPECT TO THE
RESEARCH WE'VE DONE IS WE PUT
TOGETHER A FORMAL PROVING GRANT
FUNDING PROGRAM THAT DRIVES THE
FUNDING OPPORTUNITIES AT THE
MOST PROMISING RESEARCH FOR
TREATMENT.
WE UTILIZED AN EXTERNAL BUT
PERMANENT ADVISORY COUNCIL THAT
HELPS MAKES INFORMED DECISIONS
ABOUT THE INITIATIVES IN THE
PROGRAMS.
SO SOME OF THE IDENTIFIED BROAD
RESEARCH AREAS WE'VE BEEN
HIGHLIGHTING RECENTLY ARE THE
FOLLOWING.
VERY IMPORTANT FOR US TO
IDENTIFY BIOMARKERS IN
MYASTHENIA GRAVIS AND TO PREDICT
CLINICAL OUTCOMES AND
IMMUNOSUPPRESSIVE THERAPY
RESPONSE TO UTILIZE IN CLINICAL
TRIAL.
THERE'S A NEW BIOLOGICS FOR
MYASTHENIA GRAVIS AND SOME
PATIENTS WILL RESPOND AND OTHER
SUBPOPULATIONS DON'T RESPOND.
WE DESPERATELY NEED BIOMARKERS
TO UNDERSTAND WHICH WILL WORK IN
WHICH SUBSET OF PATIENTS BEFORE
THEY'RE APPLIED.
WITH THAT THE SECOND AREA IS
DISEASE MECHANISM.
WE AIM TO UNDERSTAND THE BASIC
DISEASE MECHANISMS THAT WILL
INFORM VALUABLE BIOMARKERS IN
WHICH THERAPEUTICS WILL WORK
BEST.
THEN WE PARTNER TO LOOK FOR
TARGETED THERAPIES TO DEVELOP
NEW THERAPEUTIC TARGETS.
OFTEN TIMES IN MYASTHENIA GRAVIS
THE TREATMENT IS BROAD
IMMUNOSUPPRESSION WHICH IS
EFFECTIVE BUT HAS SIDE EFFECTS
ESPECIALLY IN THE TIME OF LIVING
THROUGH A PANDEMIC YOU DON'T
NEED OR WANT BROAD
IMMUNOSUPPRESSION TO TREAT
DISEASE WHEN THE CHANCE OF
GETTING COVID WILL INCREASE.
AND THEN WE HAVE
PATIENT-ORIENTED RESEARCH
FOCUSSED ON PATIENT OUTCOMES TO
FULLY UNDERSTAND THE IMPACT OF
THE DISEASE AND MYASTHENIA
GRAVIS DOESN'T TYPICALLY IMPACT
PEDIATRICS AND SO THERE'S A NEW
LOOK AT TREATMENT AND RESPONSE
DIFFERENTLY.
AND THE RESEARCH AWARDS THAT
HAVE COME TO LIGHT IN THE LAST
FEW YEARS ARE IMPORTANT TO
HIGHLIGHT.
I SHOULD POINT OUT RIGHT BEFORE
THE PANDEMIC STARTED THE MGFA
WAS MANAGED BY AN EXTERNAL
MANAGEMENT COMPANY AND SINCE
THEN A NEW ENTIRELY FULL-TIME
DEDICATED STAFF HAS COME ON
BOARD.
THERE'S BEEN SIGNIFICANT
INCREASE IN FUND RAISING DUE TO
THE EFFORTS OF THIS STAFF AND
NEW MANAGEMENT TEAM THAT ARE
EMPLOYEES OF THE MGFA.
AND YEARS AGO THE ONLY AWARDS
THE MGFA MADE AVAILABLE WERE
PILOT AWARDS.
WE STILL HAVE THOSE AND ADDED
OTHERS AND ADDED THEM TO
MULTIPLE AWARD SEASONS INSTEAD
OF ONCE A YEAR.
IN THE PILOT AWARDS WE'RE
LOOKING AT STUDIES TO LOOK THEY
LEAD TO NEW INVESTIGATORS.
WE ADDED IN TRANSFORMATIVE
RESEARCH AWARDS.
THESE ARE CLOSER TO WHAT AN NIH
R01 WOULD LOOK LIKE AND LOOK AT
DIRECT IMPACT ON UNDERSTANDING
MYASTHENIA AND WE HAVE
SPECIALIZED PROJECTS TARGETED TO
SPECIAL AWARDS.
FOR EXAMPLE RECENTLY THERE'S A
TYPE OF MYASTHENIA WHERE
PATIENTS HAVE THE DISEASE BUT
NOT AUTOANTIBODIES THAT ARE
DETECTABLE AND UNDERSTANDING
THAT DISEASE SUBTYPE IS VERY
IMPORTANT.
THESE PATIENTS ARE OFTEN NOT
INVOLVED IN CLINICAL TRIALS
BECAUSE THERE'S NO CLEAR
MECHANISM OF THE DISEASE.
WE FOCUS SPECIFICALLY TO
UNDERSTAND SERO-NEGATIVE
MYASTHENIA AND WE HAVE NEW
INVESTIGATOR AWARDS FOR
POST-DOCS OR CLINICIAN
SCIENTISTS STARTING THEIR
RESEARCH CAREERS AND THE IDEA IS
TO ENGAGE AND SUPPORT A DIVERSE
GROUP OF YOUNG INVESTIGATORS AND
CLINICIANS NOT ONLY TO GET THEM
INTERESTED IN UNDERSTANDING
MYASTHENIA GRAVIS BUT TO CAPTURE
THEM AND ALLOW THEM TO BUILD
CAREERS IN THIS PARTICULAR FIELD
SO THEY CAN MAINTAIN AND BUILD
LONG RESEARCH PROGRAMS.
HERE'S SOME EXAMPLES OF SOME
AWARDS WE'VE LAUNCHED IN THE
LAST COUPLE YEARS.
THE FIRST IS WE SUPPORTED WORK
FOR DIAGNOSTIC BIOMARKER AND THE
SECOND WAS A THERAPEUTIC TARGET
AND ANOTHER THAT LOOKS AT A
SUBTYPE OF MY -- MYASTHENIA AND
THE LAST ONE AWARDED TO DUKE AND
YALE IS THE SERO-NEGATIVE
PROJECT TO UNDERSTAND THE SUB
TYPE AND IMMUNOPATHOLOGY TO
UNDERSTAND HOW TO TREAT BETTER.
I WANT TO CLOSE BY POINTING OUT
THIS SUCCESS STORY WE'VE HAD AT
YALE, THIS LETTER ON THE LEFT
SIDE FROM 2013 WAS A LETTER WE
RECEIVED GIVING US A PILOT AWARD
ALMOST 10 YEARS AGO AND SIGNED
BY A GENTLEMAN WHO WAS FROM THE
PRIVATE MANAGEMENT COMPANY THAT
OVERSAW THE DAY TO DAY
OPERATIONS OF THE FOUNDATION AND
NOW IN 2022 WE HAVE A FULL-TIME
DEDICATED STAFF THAT RUNS THE
ORGANIZATION AND HAS
SIGNIFICANTLY INCREASED
DEVELOPMENT AND SUPPORT.
SINCE THIS INITIAL PILOT AWARD
IN 2013, RICH AND I BUILT AN MG
BIO REPOSITORY AND STARTED
BUILDING DATA AND A DECADE LATER
WE'VE BEEN AWARDED MULTIPLE MG
SPECIFIC GRANTS FROM THE NIH AND
R01 AND U54 PROJECT GRANT FROM
THE NINDS.
WE PUBLISHED IN OUR GROUP HERE
AT YALE ALONE OVER 20MG SPECIFIC
PUBLICATIONS STARTING IN 2013
FROM ZERO AND FOCUSSED ON MG
WITH PHARMA AND CLINICAL TRIALS
AND IMPORTANTLY THIS PROGRAM
THAT WE BUILT HERE AT YALE WITH
THE SUPPORT OF THE MGFA HAS
ALLOWED US TO TRAIN SCIENTISTS
AND CLINICIANS AND MG SPECIFIC
RESEARCH.
I'LL STOP THERE AND I'LL LOOK
FORWARD TO QUESTIONS IN THE NEXT
SESSION.
>> GREAT WORK.
THANK YOU, O'CONNOR, THANK YOU
FOR SHARING THAT WITH US.
YOU HAVE GOT A LOT DONE AND
QUICKLY.
A LITTLE BIT ABOUT THE BRAIN
DONOR PROGRAM.
WE'RE A NONPROFIT DEVELOPED OVER
SIX YEARS AGO TO SUPPORT THE
BRAIN BANKS OF THE NIH.
IT'S CALLED THE NEUROBIO BANK
AND MAKES AVAILABLE HIGH
QUALITY, DONATED HUMAN BRAIN
TISSUE TO NEUROSCIENCE
RESEARCHERS.
WE'RE ALMOST SIX YEARS OLD AND
NEARLY 6,000 GENEROUS SOULS ARE
STARTED THE PROCESS TO BECOME
BRAIN DONORS WHEN THEY DIE AND
REPRESENT NEARLY 200 CATEGORIES
OF BRAIN DIAGNOSES.
IF YOU'RE LOOKING TO FIND
ANSWERS FOR A DISEASE OR
DISORDER AND WONDERED IF YOU
NEED TO START A BRAIN BANK, SAVE
IT FOR RESEARCH AND THE BRAIN
BANK HAS AN INCREASING AMOUNT OF
CONTROLS AND ALL REQUESTABLE
ONLINE.
P.I.s FROM ALL OVER THE WORLD
GET A RESPONSE WITHIN A COUPLE
BUSINESS DAYS AND ALL THEY PAY
IS SHIPPING.
I MAY BE BIASSED BUT IT IS AMONG
THE MOST MEANINGFUL RESOURCES
KNOWN TO NEUROSCIENCE AND THE
SECOND POINT I WANT TO MAKE TO
ADVOCACY GROUPS IS THE BRAIN
DONOR PROJECT IS HAPPY TO
PROVIDE CONTENT TO EDUCATE
YOU'RE COMMUNITIES ABOUT THE
CRITICAL NEED FOR BRAIN DONATION
BY ALL KINDS OF PEOPLE.
NOT ONLY FROM THOSE WHO HAVE
BEEN DIAGNOSED BUT THEIR FRIENDS
AND FAMILY WHO CAN SUPPORT
SCIENCE BY DONATING THEIR
UNAFFECTED CONTROL BRAINS.
AS YOU KNOW THEY'RE NECESSARY IN
EVERY STUDY.
WE'LL SHARE NEWSLETTER ARTICLES
SO WEBINARS AND WRITE E-MAIL
BLASTS AND SITE COPY FOR YOUR
RESOURCE SECTION.
SAY THE WORD AND YOU CAN GET
HOLD OF ME, TISH AT BRAIN DONOR
PROJECT.org.
THERE'S QUESTIONS IN THE CHAT.
THE FIRST IS DR. IBANEZ, CAN YOU
TALK ABOUT THE CHALLENGES YOU
FACED WHEN YOU HAD TO PROVIDE
ALL RESEARCH RELATED MATERIALS
TO SPANISH SPEAKERS.
>> THANK YOU FOR THE QUESTION.
IT WAS A PROCESS.
THE MOST IMPORTANT THING WAS
TRYING TO LOCALIZE LOCAL SPOTS
IN DIFFERENT COUNTRIES WITH
RELATIVELY WELL PREPARED
RESEARCH CENTERS AND PROVIDE ALL
THE DATA FOR THE LOCAL TEAMS
FROM GENETICS TO NEURO IMAGING
AND DATA AND IN ADDITION TO THE
CONSORTIA PUBLICATION.
AND NOT ONLY PROVIDING MATERIALS
BUT TO HELP.
WE HAD TO PROVIDE SOMETIMES LIKE
THE SOFTWARE FOR THE FAMILY AND
WE ARE ABLE TO PROVIDE THAT.
WE ARE WORKING WITH THE A.D.
WORK BENCH TO PROVIDE
COMPUTATIONAL SUPPORT FOR THE
TEAM BECAUSE THEY DON'T HAVE
ENOUGH TO DEVELOP THE ANALYSIS
AND IN PARALLEL WE CREATE A KIND
OF TRAINING PROCESS.
WE TRAIN PEOPLE IN CLINICAL
ASSESSMENT AND CREATE DIFFERENT
TUTORIALS AND COURSES FOR
CLINICAL ASSESSMENT AND THAT'S
OUR CHECKLIST AND WE ARE TRYING
TO EMPOWER THE LOCAL TEAMS AND
CONNECTING WITH THE CONSORTIA
AND GIVING THE OPPORTUNITY FOR
THE DIFFERENT CENTERS TO MAKE
THE SPECIFIC RESEARCH.
IN ADDITION AND INCLUDING THE
SPECIAL TASK FOR QUESTION AREA
AND THEY CAN USE ALL THE DATA
PROVIDED.
WE ARE TRYING TO PROVIDE
HOLISTIC SUPPORT FOR THE TEAMS.
>> HOLISTIC IS RIGHT.
THAT'S A LOT.
AND YOU STARTED TO ANSWER SOME
OF THE NEXT QUESTION WHICH WAS
ALSO ASKED ABOUT ENTICING
PROFESSIONALS TO ATTEND THE
TRAININGS.
DR. BECK, I SEE YOU OFFERED A
RESPONSE.
CAN YOU TALK ABOUT THAT A LITTLE
BIT.
>> ABSOLUTELY.
SO TRYING TO BRING PEER
PROFESSIONALS IN THE PROCESS WAS
THREE NETWORKING AND STEWARDSHIP
WITHIN THE U.S. AND WE ACADEMIC
MEDICAL CENTERS AND WE ASKED
INDIVIDUALS TO REACH OUT IT
COMMUNITIES TO FIND PROMOTORES
AND WE RECEIVED A GENEROUS GRANT
AND PROVIDE STIPENDS.
THERE'S AN ENGAGEMENT BODY AND
WE RECOGNIZED THERE'S NOT ENOUGH
PEOPLE WHO WHETHER THEY'RE
CLINICIANS OR GENETIC COUNSELORS
KNOW ENOUGH ABOUT PARKINSON'S
GENETICS AND TRYING TO PROVIDE
CME CREDITS TO LEARN MORE ABOUT
PARKINSON'S AND HOW THE
DISCUSSIONS SHOULD BE STRUCTURED
IN RETURNING RESULTS TO PEOPLE
WITH PD.
>> YOU TALKED ABOUT AN
ACCREDITATION BODY FOR
PROMOTORES.
>> THERE'S A CREDENTIALING BODY
AND OUR FOUNDATION IS ABLE TO
PROVIDE ACCREDITATION FOR CMEs
AND CEUs AND LOOKING TO GET
OURSELVES CERTIFIED TO DO THE
SAME FOR COMMUNITY HEALTH
WORKERS SO THE ADDITIONAL
INCENTIVE WE OFFERED IT FOR FREE
OF COURSE AND BECAUSE THE
PROFESSIONALS NEED THESE
CONTINUED EDUCATION CREDITS
THERE'S A REAL INCENTIVE TO
PARTICIPATE IN SOMETHING THAT'S
FREE AND COMMUNITY HEALTH
WORKERS WHERE THEY MAY HAVE TO
DO IT OUT OF THEIR OWN POCKET
AND MORE INCENTIVE TO LEARN
ABOUT RESEARCH AND PARKINSON'S
DISEASE.
>> THAT'S A SUCCESS STORY IN
ITSELF TO LEARN THE RESOURCES
HELP ALL OF US AND ACCEPT
INVITATIONS ON A VERY LOCAL
LEVEL.
THAT'S BRILLIANT.
ANY OTHER QUESTIONS FROM THE
GROUP?
I'M SEEING A QUESTION ABOUT
BRAIN DONATION.
I'LL CIRCLE BACK.
WE DO SPINAL FLUID AND OTHER
THINGS AND WE'LL WORK WITH
DIFFERENT GROUPS WHO NEED
SPECIFIC TISSUES FROM TIME TO
TIME.
>> TISH, I WANT TO MAKE ANOTHER
VOTE FOR BRAIN DONATION IN THE
PARKS PARKINSON'S COMMUNITY IT'S
BEEN CRITICAL TO ADVANCE
RESEARCH FOR PEOPLE WHO MAKE
THAT GENEROUS DONATION OF TISSUE
AT THEN OF LIFE.
IT'S HOW WE'VE BEEN ABLE TO MAKE
KEY ADVANCEMENTS.
PARKINSON'S DISEASE HAPPENS IN
PEOPLE AND NOT ANIMALS.
>> WE HAVE ALMOST 2,000 PEOPLE
WHO REPORTED PARKINSON'S
DIAGNOSIS WHO PLAN TO DONATE
THEIR BRAINS.
IT SEEMS THERE'S BEEN A LOT OF
QUESTION AND ENGAGING UNDER
SERVED POPULATIONS WHICH IS
SOMETHING WE'RE ALL WORKING
HARDER TO DO RIGHT NOW.
VERY VALUABLE ESPECIALLY TO ME
TO UNDERSTAND THE RESOURCES OUT
THERE FOR THAT ENGAGEMENT.
DR. IBANEZ, THAT'S THE MOST
COMPLICATED WORK WITH PEOPLE IN
DIFFERENT COUNTRIES AND CULTURES
AND DIFFERENT SOCIAL
DETERMINATES OF HEALTH.
I APPLAUD YOUR ORGANIZATION FOR
WHAT YOU'RE ABLE TO GET DONE AND
WILL HELP MOVE SCIENCE FORWARD.
DR. BECK, I LEARNED SO MUCH
ABOUT PARKINSON'S AND HAD NO
IDEA THE GENETIC COMPONENT WAS A
SMALL MINORITY OF CASES BUT SO
CRITICAL TO UNDERSTAND THAT A
LITTLE BIT BETTER AND
DR. O'CONNOR, WE HAVE A BOARD
MEMBER WITH MG AND I UNDERSTAND
MORE ABOUT WHAT HE STRUGGLES
WITH FROM TIME TO TIME.
SOMETIMES AT THE LAST MINUTE HE
CAN'T ATTEND A MEETING BECAUSE
HIS VERBAL IS VERY IMPACTED BY
THIS CONDITION.
YOU EDUCATED ME.
AND IF ANYONE HAS QUESTIONS WE
HAVE A COUPLE MORE MINUTES TO
MOVE FORWARD.
DO ANY OF OUR PANELISTS FEEL
LIKE THERE'S SOMETHING THEY
WANTED TO SAY THEY DIDN'T GET A
CHANCE TO
BECAUSE WE CAN CIRCLE
BACK IN THE ABSENCE OF ANY
FURTHER QUESTIONS?
>> I KNOW THERE'S A QUESTION
GERALDINE ASKED ABOUT SPANISH
MATERIALS.
I ALSO WANT TO MENTION FOR OUR
STUDY AS PARTS OF OUR OUTREACH,
FOR PEOPLE WHO ARE INTERESTED IN
TRYING TO ENGAGE SPANISH
SPEAKERS IN THE U.S. THROUGH THE
STUDIES, IT'S NOT ALWAYS A
STRAIGHTFORWARD PROCESS.
TRANSLATION IS JUST PART OF IT
BUT IDENTIFYING INDIVIDUALS WHO
CAN THEN REACH OUT TO THE
COMMUNITY IS PART OF IT AND THE
OTHER SIDE AND I THINK AGUSTIN
WAS TALKING ABOUT THIS WITH HIS
OUTREACH WE SEE ISSUES AROUND
SOCIO ECONOMIC STATUS AND TRYING
TO GAIN ACCESS TO SOME
RESOURCES.
WE LAUNCHED OUR STUDY IN THE
WINTER OF 2019 AND THEN COVID
CAME IN MARCH AND WE HAD TO
QUICKLY PIVOT TO A TELEMEDICINE
APPROACH AND WERE ABLE TO WHICH
IS GREAT AND YOU CAN DO GENETIC
COUNSELLING AND A BUCKLE SWAB AT
HOME TO COLLECT DNA SAMPLES AND
MANY MEMBERS OF OUR
SPANISH-SPEAKING COMMUNITY
DIDN'T HAVE ACCESS TO RESOURCES
AND HAD LOTS OF QUESTIONS.
IT'S DIFFERENT THAN WHEN WE'RE
JUST DEALING WITH WHITE COLLARED
EDUCATED MEN THE DOMINANT
PARTICIPANTS IN RESEARCH.
IT REQUIRES MORE EFFORT AND
PROMOTORESES ARE A GREAT PART OF
THAT AND I HAVE MEMBERS ON MY
TEAM WHO ARE SPANISH SPEAKERS,
DOMINICAN REPUBLIC AND PUERTO
RICO AND IN GENETICS IS ABOUT A
FAMILY AND MORE PEOPLE MAY BE
INVOLVED IN THE CONSENTING
PROCESS AND THE DISCUSSION ABOUT
THE RETURN ON RESULTS.
BEING ABLE TO BE FLEXIBLE AROUND
THAT IS IMPORTANT TO SEE
SUCCESSFUL ENGAGEMENT IN
COMMUNITIES OTHER THAN THE EURO
ETHNIC COMMUNITIES WHICH
DOMINATE OUR STUDIES.
IT'S NOT JUST THE SOCIAL JUSTICE
ISSUE THERE'S SO MUCH TO BE
LEARNED SCIENTIFICALLY FROM
PEOPLE FROM DIVERSE BACKGROUNDS
AND IN PRECISION MEDICINE IT'S
IMPORTANT TO HAVE PEOPLE
REPRESENTED SPECIFICALLY AROUND
GENETICS TO BE INCLUDED AS PART
OF THIS GENETIC, PRECISION
MEDICINE REVOLUTION.
PEOPLE OF COLOMBIAN DESCENT HAVE
A UNIQUE MUTATION THAT CAN LEAD
TO PARKINSON'S DISEASE AND YOU
CAN SEE A COMPANY MAKING A
THERAPEUTIC AND LIST WHAT GENES
AND PERHAPS MUTATIONS AND IF
SOMEONE'S LEFT OFF THEY MAY NOT
GET ACCESS TO THE TREATMENT OR
INSURANCE COVERAGE.
THESE ARE THE THINGS THAT ARE
ADD-ONs IT'S IMPORTANT TO ENSURE
EVERYONE IS PART OF THIS.
THERE'S MANY A STONE TO BE
UNTURNED FOR HELPING THE DISEASE
AND COMMUNITIES AT LARGE.
>> EXCELLENT POINTS.
THANK YOU FOR SHARING THEM.
ONE MORE QUESTION TO
DR. O'CONNOR.
DR. KOROSHETZ IS WONDERING ABOUT
THE FOUNDATION'S THOUGHTS ON THE
NEW MEDICATION FOR ACHRAB
POSITIVE PATIENTS.
HE'S WONDERING HOW THAT BECAME A
REALITY AND DID THE FOUNDATION
PLAY A ROLE IN THAT?
>> WHICH PARTICULAR MEDICATION
ARE YOU SPEAKING ABOUT WALTER?
>> I THINK THE INHIBITOR.
>> OH, YEAH, SURE.
MY LAB DOES SOME WORK ON THAT.
I WOULDN'T SAY THE FOUNDATION
WAS DIRECTLY INVOLVED IN THAT
DEVELOPMENT AT THIS POINT.
I THINK THE FOUNDATION MADE A
TURNING POINT IN THAT WE DO HAVE
MORE ESTABLISHED COLLABORATIONS
WITH CLINICAL TRIALS THAT ARE
ONGOING AND SUPPORTED BY
PHARMACEUTICAL COMPANIES.
THE COMPLIMENT INHIBITOR YOU'RE
SPEAKING ABOUT IS ONE OF THE
EXAMPLES OF THE NEW BIOLOGICS
THAT HAVE BECOME AVAILABLE THAT
HAVE VERY SURPRISING RESULTS.
WHAT I MEAN BY THAT IS IF THE
PATIENTS THAT ARE TAKING IT HAVE
ANTIBODIES AND THERE'S A SUB
POPULATION IN PATIENTS THAT
DON'T RESPONSE OR RESPOND LATE
THAT PARTICULAR THERAPEUTIC AND
THE FOUNDATION IS SUPPORTING
RESEARCH TO FURTHER INVESTIGATE
WHY THERE ARE HETEROGENEITY OF
PATIENTS THAT RESPOND TO THIS
DRUG IN A WAY THAT'S UNEXPECTED.
I GUESS IT'S AN EXAMPLE OF WHY
IT'S IMPORTANT TO PARTNER NOT
ONLY WITH ACADEMIC LABORATORIES
THAT CAN DO THIS KIND OF
RESEARCH BUT THERE NEEDS TO BE
PARTNERSHIP WITH PHARMA AS WELL
BECAUSE THEY'RE RUNNING THE
TRIAL AND HAVE THE PATIENT
SAMPLES FROM THOSE THAT RESPOND
AND THOSE THAT DON'T RESPOND.
IN ONE OF THE EXAMPLES I HAVE,
THE MGFA IS SUPPORTING RESEARCH
TO EXAMINE SPECIFICALLY THOSE
SAMPLES THAT HAVE COME FROM A
CLINICAL TRIAL OF A COMPLIMENT
INHIBITER LOOKING AT PATIENTS
THAT RESPOND AND PATIENTS THAT
DON'T TO FIND OUT WHY SO WE CAN
AVOID THIS VERY EXPENSIVE DRUG
THAT TAKES A LONG TIME TO TAKE
BEFORE IT'S GIVEN TO SOMEBODY
BEFORE KNOWING WHETHER IT'S
GOING TO WORK OR NOT.
>> I WAS GOING TO ASK -- SO
NINDS OFFERS GLOBAL HEALTH AND
RICHARD BENSON RUNS THAT OFFICE.
WE HAVE A STRATEGY OF WORKING
THROUGH THE FOGARTY INSTITUTE
BECAUSE THEY HAVE CONTACTS ALL
OVER AND THE BRAIN DISORDER
DEVELOPING WORLD WE FUND.
IT'S RUN THROUGH THE FOGARTY AND
WE RUN THE PROJECT THAT ARE
NEU
NEURO BASED AND WE HAVE SOME
LIKE TRAUMATIC BRAIN INJURY AND
IS THE FOGARTY INSTITUTE WELL
KNOWN WITH YOUR GROUND?
DO YOU WORK WITH THEM?
>> QUESTION.
>> YES.
THANK YOU.
WE WOULD LIKE TO CREATE A
REGISTRY.
TRAINING PEOPLE AND CREATING
QUALITY CHECK TRAININGS.
WE HAVE BEEN WORKING A LOT
TOWARDS THE INITIATIVE.
I THINK WE CAN TRANSLATE THIS
INITIATIVE TO OTHER FIELDS AND
DISORDERS.
I'M LOOKING FORWARD TO LEARN
MORE ABOUT THIS AND HAPPY TO
EXPLORE ANY OPPORTUNITY FOR
THAT.
>> THE LINK WAS JUST POSTED IN
THE CHAT.
I THINK WE'RE OUT OF TIME.
IT'S BEEN FASCINATING FOR ME AND
I KNOW IT HAS BEEN FOR OTHER
PEOPLE AND I WANT TO THANK THE
PANELISTS FOR THE BRILLIANT WORK
YOU'RE DOING AND TAKING THE TIME
TO SHARE IT.
THANK YOU, MUCH.
>> THANK YOU SO MUCH.
>> BYE-BYE.
>> GREAT DISCUSSION.
THANK YOU, TISH.
AND NOW WE'RE GOING TO MOVE INTO
THE SECOND TOPIC FOR THE
MORNING, WHICH IS ABOUT HOW TO
WORK TOGETHER TO ACHIEVE EQUITY
AND INCLUSIVITY IN THE RESEARCH
AND ALSO IN THE INVOLVEMENT OF
PATIENTS IN DISEASE GROUPS.
CERTAINLY AT NIH WE'VE BEEN
STRUGGLING WITH THIS PROBLEM FOR
MANY YEARS.
I THINK WE MADE SOME HEADWAY BUT
SEEMS TO BE AN UP HILL BATTLE.
WE HAVE LEARNED WHEN IT COMES TO
RESEARCH THERE'S NOT INNATE
TRUST IN AND IT TAKES A LONG
TIME TO BUILD THAT UP.
AND THE SITUATION OF TRYING TO
HELICOPTER IN THE RESEARCH GROUP
TO TRY TO GET SOMETHING DONE AND
OUT IS NOT FAIR FOR THE
COMMUNITY AND WE'RE TRYING TO
LOOK AT COMMUNITIES TO DO
RESEARCH.
I'M INTERESTED TO HEAR FROM THE
DISEASE ORGANIZATIONS HOW TRUST
BUILDING HAS WORKED IN YOUR
AREAS AND MAYBE LESSONS LEARNED
FROM NIH TO FOLLOW AS WELL.
SO WE'RE HAPPY TO HAVE JANET
FROM THE DYSTONIA MEDICAL
RESEARCH FOUNDATION LEADING THE
GROUP AND LOOKING FORWARD TO
WHAT PEOPLE HAVE TO SAY FROM
THEIR EXPERIENCES.
LET ME TURN IT OVER TO YOU,
JANET.
IS THAT OKAY?
>> GREAT, THANK YOU, WALTER.
I APPRECIATE IT.
I WANT TO THANK YOU FOR YOUR
ONGOING COMMITMENT TO THE
NONPROFIT FORUM TO BRING US ALL
TOGETHER TO LEARN FROM ONE
ANOTHER.
AND MANY THANKS TO THE TEAM AT
NINDS AND SAMANTHA AND ANDREA
WHO HAVE BEEN WONDERFUL IN THEIR
EFFORTS AND THANK YOU TO
HEATHER, CAMERON AND THE TEAM AT
INFINITY CONFERENCES.
WE HAVE DONE THESE MEETINGS AND
LOTS OF MOVING PARTS.
WELL DONE, EVERYBODY.
THANK YOU FOR THE OPPORTUNITY.
THE SESSION IS THE ENTIRE
COMMUNITY HOW TO WORK TO ACHIEVE
EQUITY AND INCLUSIVITY.
I THINK IT'S NEAR AND DEAR TO
ALL OUR HEARTS.
WORKING TO ENGAGE ALL THE
STAKEHOLDERS IS REALLY IMPORTANT
AS WALTER SAID THIS MORNING.
NOT JUST THOSE AT THE FRONT OF
THE LINE BUT ABSOLUTELY
EVERYBODY IN OUR REPRESENTATIVE
DISEASE COMMUNITIES IS CRITICAL
TO OUR BEING ABLE TO BE
SUCCESSFUL AND SUPPORTIVE OF THE
NEUROLOGICAL DISEASES.
I BELIEVE PATIENT ORGANIZATIONS
HAVE BEEN A MAJOR FACTOR.
A DRIVING FORCE ON THIS
IMPORTANT TOPIC WORKING WITH
RESEARCHERS AND INSTITUTIONS TO
HELP RAISE THEIR AWARENESS THAT
ALL PATIENT VOICES NEED TO BE
HEARD.
I THINK JIM BECK'S PRESENTATION
IS A GOOD EXAMPLE OF HOW PATIENT
ORGANIZATIONS CAN HAVE A HUGE
IMPACT ON THIS AREA AND WE HAVE
A TERRIFIC PANEL TODAY TO SHARE
WITH US THEIR EXPERIENCES AND
REACHING OUT TO RARE DISEASE
COMMUNITIES.
THEY'RE COMPLETE BIOS ARE ON THE
WEBSITE FOR THE FORUM AND I
ENCOURAGE YOU TO TAKE A LOOK AT
THOSE BUT I DO WANT TO BRIEFLY
INTRODUCE OUR PANEL MEMBERS AND
THEN WE'LL HAVE PRESENTATIONS
AND THEN DO AS MANY QUESTIONS AS
TIME WILL ALLOW.
I ENCOURAGE YOU TO USE THE CHAT
FUNCTION TO PUT YOUR QUESTIONS
IN THERE AS YOU THINK OF THEM SO
YOU DON'T FORGET THEM.
WE'LL HEAR FIRST FROM DR. DEL
PILAR TRELLES WITH EXPERTISE IN
AUTISM SPECTRUM DISORDER AND
ASSISTANT PROFESSOR IN
PSYCHIATRY AND HUMAN BEHAVIOR AT
THE CENTER FOR AUTISM AND
DEVELOPMENT DISABILITIES IN
RHODE ISLAND.
AND HEAR FROM DR. SCHWEDT A
PROFESSOR OF NEUROLOGY AND VICE
CHAIR FOR NEUROLOGY RESEARCH AND
DIRECTOR OF THE UNIT AT MAYO
CLINIC IN ARIZONA AND SERVES ON
THE EXECUTIVE COMMUNITY OF THE
BOARD FOR HEADACHE SOCIETY AND
MIGRAINE FOUNDATION.
HEATHER WATKINS IS A DISABILITY
ADVOCATE, AUTHOR, BLOGGER, WOMAN
OF MANY TALENTS WHO PROVIDES HER
EXPERTISE TO COMMUNITY AND
GOVERNMENT ORGANIZATIONS AS A
CONSULTANT AND LOOK AT HER BLOG,
SLOW WALKERS SEE MORE.
WONDERFUL READ AND IMPORTANT
INSIGHTS AND LAST BUT NOT LEAST
WE WILL HEAR FROM INDU NAVAR WHO
FOLLOWING THE DEATH OF HER
HUSBAND FROM ALS IN 2019 HAS
DEDICATED HERSELF TO USING THE
EXPERTISE AND KNOWLEDGE GAINED
AS A SILICON VALLEY TECH
EXECUTIVE TO DEVELOP TECHNOLOGY
BASED SOLUTIONS TO THE DISCOVER
OF BIO MARKERS AND NEUROLOGICAL
DISEASES AND CEO AND FOUNDER OF
EVERYTHING ALS AND SERVES ON THE
ADVISORY BOARD OF THE ANSWER ALS
ORGANIZATION.
WONDERFUL EXPERTS TO SHARE
LEARNINGS WITH US THIS MORNING.
>> I FATHER WAS A NEUROLOGIST
AND WOULD DO ROUNDS ON THE
WEEKENDS AND THIS IS THE
HOSPITAL AND AS A LITTLE GIRL
I'D LIKE TO GO WITH HIM TO THE
HOSPITAL TO SPEND TIME WITH HIM
AND EARLY ON I LEARNED THAT WE
NEED IN A WORLD OF CONTRAST THE
HOSPITAL AS BEAUTIFUL AS IT IS
SITS IN ONE OF THE POOREST
NEIGHBORHOODS IN LIMA.
WHEN YOU HAVE THAT CONTRAST YOU
ALSO HAVE A LOT OF HEALTH CARE
DISPARITIES.
WHEN I FIRST CAME TO THE U.S. TO
PURSUE MY RESIDENCY TRAINING I
QUICKLY REALIZED THAT THOSE
HEALTH DISPARITIES WERE PRESENT
IN THE U.S. AND MORE STRIKING
GIVEN THE AMOUNT OF RESOURCES
THAT WE HAVE.
AFTER I FINISHED MY CLINICAL
TRAINING I DECIDED TO EMBARK ON
A CAREER OF RESEARCH AND GOT MY
FIRST GRANT COMING FROM THE
CLINICAL WORLD AND WAS VERY
EXCITE
EXCITED AND WE HAD PAIRED
CAREGIVERS WITH ASD WITH KIDS OF
FAMILIES WHO WERE DIAGNOSED TO
LEARN HOW TO NAVIGATE THE
COMPLEX HEALTH CARE SYSTEM.
I WAS EXCITED AND IT DIDN'T WORK
AND FAMILIES DIDN'T STICK AND IT
WAS A HUMBLING EXPERIENCE BUT
ONE THING WE LEARNED AS WE
LOOKED AT THE DATA WAS THE
FAMILIES THAT DID STAY WERE THE
FAMILIES SOMEHOW CONNECTED TO
THE MOUNT SINAI HEALTH SYSTEM
WHERE I WAS.
THE FIRST LESSON IS IF YOU WANT
TO REACH COMMUNITIES, YOU NEED
TO BE IN THE COMMUNITY.
YOU NEED TONE TO BE PART OF THE
COMMUNITY AND I HAVE THIS SLIDE
AS A REPRESENT SOME OF THE WORK
WITH THEM BECAUSE I WANT YOU TO
KNOW THIS IS A MODEL THAT TRULY
WORKS.
IN THREE YEARS WE CHANGED THE
DEMOGRAPHICS OF THE FAMILIES WE
WERE SEEING AT THE AUTISM CENTER
WHICH IS A RESEARCH CENTER TO A
GRAPHIC THAT WAS MORE SIMILAR TO
THE LOCATION WHERE THE MOUNT
SINAI HOSPITAL SITS.
THIS IS A WELL DEFINED PUBLIC
HEALTH MODEL THAT LEVERAGES THE
RESOURCES BROUGHT IN BY ACADEMIC
INSTITUTIONS TO THE BENEFIT OF
ALL.
CENTRAL TO THIS IDEA IS THE
THERE'S BI-DIRECTIONAL LEARN AND
RESEARCHERS AND COMMUNITY
MEMBERS LEARN FROM ONE ANOTHER.
THEY WORK AS A TEAM.
THIS IS A GRAPHIC REPRESENTATION
OF HOW IT WOULD LOOK AND LOOKED
AT THE RARE DISEASE CLINIC
RESEARCH NETWORKS AND THEY WERE
STRUGGLING TO REACH FAMILIES
THAT COME FROM UNDER REPRESENTED
AND UNDER RESOURCED COMMUNITIES.
WE BASICALLY BROUGHT THEM IN THE
RESEARCH TEAM AND THAT'S WHAT
THIS MODEL IS ABOUT AND INCLUDED
FAMILY MEMBERS AND COMMUNITY
CLINICIANS IN THE RESEARCH TEAM
AND THAT'S HOW THE RESEARCH
STUDY WAS SET UP.
AND THERE'S SUSTAINABILITY AND
IN MANY WAYS IT'S THE HARDEST
PART.
IN THE INTEREST OF TIME I'M
GOING TO TALK TO YOU ABOUT
PARTNERSHIP THAT I FORMED WITH
THE CENTER WHICH SHOWS THE
BEAUTY OF THE MODEL.
THERE'S A NON-PROFIT IN LIMA
THAT SERVES KIDS WITH DIFFERENT
ACCOUNTABILITIES AND BELIEVED IN
THE POWER OF TWO.
AND IN ORDER TO SUPPORT CHILDREN
WITH DEVELOPMENT DISABILITIES WE
NEED TO WORK WITH FAMILIES AS A
TEAM.
AND AT FIRST WE WERE BRINGING
OUR EXPERTISE.
IT STARTED AN THEY WERE OVER
BURDENED WITH CLINICAL CARE AND
THE LAST THING THEY WOULD WOULD
HAVE THOUGHT POSSIBLE AND I
THINK PART LY THE WORK WAS
AMAZING AND INSPIRATIONAL AND WE
START SOMETHING WONDERFUL
HAPPENED.
WE GAVE AIM SEMINAR ON THE
GENETICS AND GENOMICS IN AUTISM
CARE AND HOW THAT WAS SHAPING
THE FIELD.
SOMETHING SOMETHING THAT WOULD
HAVE BEEN IN THOUGHT OF ENGAGING
FAMILIES IN RESEARCH AND IT WAS
THIS PERFECTLY OIL MACHINE.
WE HAD AN IRB AND WE RECRUITED
100 FAMILIES AND WE SAW 40
FAMILIES RAISING 45 CHILDREN
WITH AUTISM AND MULTIPLE
DISABILITIES.
AND WE DID GENETIC TESTING AND
ADMINISTERED A NUMBER OF
CLINICAL MEASURES AND BECAUSE
YOU WANT TO BALANCE THE NEEDS OF
THE COMMUNITY WE ADVOCATED TO
GIVE THEM BACK CLINICAL GENETIC
RESULTS.
IT WAS SUPPORTED BY THE AUTISM
SEQUENCING CONSORTIUM.
WE FOUND THIS COMMON GOAL AND
WORKED TOGETHER.
I THINK WE SAW THE POWER WHEN
THE COVID-19 PANDEMIC HIT.
AND WE DIDN'T DO WELL WITH
FAMILIES STRUGGLING AND THE
FAMILIES IN PERU WERE DOING
AMAZINGLY WELL AND THERE WAS A
WONDERFUL MODEL WORKING WITH
FAMILIES TRANSITIONING INTO
LEARNING AND WE SAID PERU, WE
NEED YOUR HELP.
AND WE JUST FINISHED THE STUDY
AND WERE SEEING WONDERFUL
IMPROVEMENT AND CHILD AND
CAREGIVER AND I COULD TALK ABOUT
THAT FOR HOURS AND I THINK THESE
WORDS ENCAPSULATES WHAT DOING
THIS KIND OF WORK DOES.
>> I THINK SHE ENCAPSULATES WHAT
THIS IS ABOUT AND IT'S PROVEN TO
BE SUCCESSFUL TO INCREASE THE
RELEVANCE AND IMPACT OF THE WORK
WE DO.
IT DOES FEEL LIKE AN UPHILL IN
THE PROJECT AND IT'S TAKEN ABOUT
10 YEARS TO GET HERE BUT THE
REWARDS HAVE BEEN IMMENSE AND I
BELIEVE IN THIS TYPE OF MODEL
LIE
LIES WHAT WE NEED TO ADDRESS
HEALTH CARE AND HOPE YOU'LL
CONSIDER IT IN THINKING OF YOUR
FUTURE RESEARCH PROJECTS.
I'D LIKE TO THANK EVERYONE WHO'S
BEEN PART OF THIS AND IT
REQUIRES THE SUPPORT OF A LOT OF
PEOPLE.
I DIDN'T WANT TO END WITHOUT
THAT.
>> FIRST, CONGRATULATIONS.
WHERE HE LOOK FORWARD TO
PRESENTING QUESTIONS.
DR. TODD SCHWEDT.
>> IT'S AN HONOR TO BE PART OF
THE MEETING THE LAST TWO DAYS.
I'LL APOLOGIZE UP FRONT.
I'M A LITTLE UNDER THE WEATHER.
I APOLOGIZE FOR MY VOICE IF I
END UP COUGHING.
I'LL TALK ABOUT THE IMPORTANCE
OF INCLUDING PATIENT AS ETHICAL
PARTNERS IN CLINICAL RESEARCH.
I WANTED TO START BY LOOKING AT
PCORI AND WHAT IS PATIENT
ENGAGEMENT IN RESEARCH.
IT SAYS IT'S THE MEANINGFUL
INVOLVEMENT OF PATIENTS
THROUGHOUT THE ENTIRE RESEARCH
PROCESS FROM PLANNING THE STUDY
TO CONDUCTING THE STUDY AS WELL
AS DISSEMINATING THE STUDY
RESULTS.
IT'S VERY CLEAR IT LEADS TO MORE
SUCCESSFUL PATIENT AND RESEARCH
PROJECTS THEREFORE YIELDS MORE
IMPACTFUL RESULTS.
IT'S MY BELIEF THAT RESEARCHERS
AND SCIENTISTS SHOULD NO LONGER
BE DETERMINING THE MOST
IMPORTANT CLINICAL RESEARCH
QUESTIONS OR OUTCOMES OR METHODS
IN ISOLATION BUT WITH PATIENT
PARTNERS.
SO JUST A RULE FOR MYSELF, I
BELIEVE IF WE'RE ASKING PATIENTS
TO PARTICIPATE IN SUBJECTS WE
SHOULD INCLUDE PATIENTS AS
EQUITABLE RESEARCH PARTNERS.
I JUST WANT TO TALK ABOUT OUR
RECENT EXPERIENCES AS INVOLVING
PATIENTS AS EQUITABLE RESEARCH
PARTNERS.
IT'S PART OF A PRAGMATIC
RANDOMIZED PROSPECTIVE CLINICAL
TRIAL CALLED THE MEDICATION
OVERUSE TREATMENT STRATEGY
TRIAL.
IT'S A PCORI TRIAL AND ENROLLED
PATIENTS FROM ALL OVER THE
UNITED STATES AND COMPARED TWO
TREATMENT STRATEGIES FOR PEOPLE
WHO HAVE WHAT'S CALLED CHRONIC
MIGRAINE IT REFERS TO THOSE
INDIVIDUALS WHO HAVE HIGH
FREQUENCY MIGRAINE 15 OR MORE
HEADACHE DAYS PER MONTH AND AT
LEAST EIGHT DAYS PER MONTH WITH
THE FULL-BLOWN SYSTEMS AND MEET
THE CRITERIA FOR MEDICATION
OVERUSE AND END YOU HAVE NEEDING
ADD-NEEDED MEDICATION TO TREAT
SYMPTOMS MORE FREQUENTLY THAN
PRE-DEFINED THRESHOLDS.
IN THE PLANNING STAGE WE
DISCUSSED THE IDEA AT A NATIONAL
PATIENT MEETING AND HAD SURVEYS
AT IN-PERSON MEETINGS AND HAD
FACEBOOK GROUPS AND FOCUS GROUP
MEETINGS WITH PEOPLE WHO HAVE
THE DISEASE THAT WE WERE GOING
TO STUDY AS WELL AS THEIR
CAREGIVERS.
WE WANTED TO ADDRESS THE ISSUE
ABOUT THE DISEASE AND WHICH
OUTCOMES ARE MOST MEANINGFUL AND
RELEVANT AND THROUGH DISCUSSION
AND SURVEYS AND PATIENTS WERE
RANKING WHAT PRIMARY AND
SECONDARY OUTCOMES THEY FELT WE
SHOULD LOOK AT AND ALL WAS DONE
WITH THE HELP OF A PATIENT
CO-INVESTIGATOR AND THERE WAS
ONE CO-INVESTIGATOR WHO WAS A
PATIENT PARTNER, TERRY ROBERT.
>> THEN MOVING TO THE NEXT PHASE
OUR PATIENT PARTNERS HELP WITH
WRITING MATERIALS AND OUR
PATIENT CO-INVESTIGATOR FILMED A
VIDEO OF HERSELF DESCRIBING THE
STUDY IN PATIENT-FRIENDLY TERMS
AND WAS SENT TO THE SIGHT AND
HAD THE OPTION TO USE IT AND
SHOW IT TO POTENTIAL
PARTICIPANTS AS THEY WERE
CONSIDERED WHETHER OR NOT TO
PARTICIPATE IN THE CLINICAL
TRIAL.
PATIENT PARTNERS HELPED THROUGH
VIEWING AND EDITING
QUESTIONNAIRES AND HEADACHE
DIARIES AND SERVED ON THE
STEERING COMMITTEE AND DSMB.
THEY CO-PRESENTED AT ANNUAL
UPDATE MEETINGS OFTEN TIMES
CALLED ANNUAL INVESTIGATOR
MEETINGS AND THROUGH THE
PARTICIPATION WE ADMINISTERED
SURVEYS THEY CAN ANSWER
ANONYMOUSLY.
IT A SURVEY THAT WOULD ASSESS
THEIR PERCEPTION OF HOW INVOLVED
THEY WERE IN WHETHER OR NOT THEY
WERE BEING TREATED EQUITABLY.
IF THERE'S ROOM FOR SUGGESTIONS
FOR IMPROVEMENT WE'D ACT TO MAKE
THOSE IMPROVEMENTS.
OUR PATIENT PARTNERS HELP.
IT ENDS UP THE TERM MEDICATION
OVERUSE SO THAT'S THE OFFICIAL
TERM FOR PEOPLE USING ADD-NEEDED
MEDICINES ABOVE A FREQUENCY
THRESHOLD AND ENDS UP FOR SOME
PATIENTS AND CLINICIANS THE TERM
MEDICATION OVERUSE HAS A
NEGATIVE CONNOTATION.
IT SOUNDS LIKE WE'RE BLAMING THE
PATIENT FOR THEIR OWN CONDITION.
AS THE NOT THE INTENT AND OUR
PATIENT PARTNERS HELPED TO
REFRAME THE STUDY AND THINK OF
BETTER WAYS MORE ACCEPTABLE WAYS
TO DESCRIBE THE DISEASE WHEN
WE'RE ASKING PATIENTS TO
PARTICIPATE IN THE STUDY.
AND THERE'S THE IMPORTANCE OF
WHAT WE WERE DOING AND THE
IMPORTANCE OF FINDING THE
RESULTS.
WE HAD TO CHANGE OUR SAMPLE SIZE
DURING THE STUDY WHICH MEANT WE
HAD TO CHANGE OUR NON
INFERIORITY PROBLEM AND CHANGING
THE NON-INFERIORITY MARGIN WOULD
NOT CHANGE THE STUDY.
THEY HELPED DISSEMINATE RESULTS
AND HAD THEM PRESENT DATA AT
CONFERENCES.
THEY REGULARLY SHARED PROGRESS
AND RESULTS IN SOCIAL MEDIA AND
CONTRIBUTED TO THE STUDY WEBSITE
AND NEWSLETTER.
A PERSON SAYS BEING A PATIENT
ADVOCATE LET ME SEE HOW CRUCIAL
IT IS TO PATIENTS AND PARTNERS
AND HELPED THE RESEARCH WORK
BETTER AND FELT DEEPLY INVOLVED.
AS SOMEONE ONE MIGRAINE DISEASE
THE EXPERIENCE GAVE ME GREAT
HOPE FOR BETTER TREATMENTS FOR
THOSE OF US WHO SUFFER WITH THIS
CONDITION.
I HOPE TO WORK ON MORE MIGRAINE
RESEARCH TRIALS IN THIS
CAPACITY.
FEEL FREE TO REACH OUT WITH
QUESTIONS OR COMMENTS AT MY
E-MAIL ADDRESS THERE.
>> THANK YOU.
NEXT WE'LL HEAR FROM HEATHER
WATKINS.
WE HAVE A RE-RECORDED VIDEO.
SHE'LL BE WITH US LIVE FOR Q&A.
>> I'M A BOSTON-BASED ADVOCATE
AND MOTHER AND AUTHOR AND
BLOGGER AND A COMMUNITY BUILDER.
I SERVE ON A HANDFUL OF
DISABILITY AND RELATED BOARDS
AND CONTRACTS INCLUDING AS A
FORMER CHAIRPERSON FOR THE
BOSTON MAYOR'S COMMISSION FOR
PERSONS WITH DISABILITIES
ADVISORY BOARD.
I WAS ABLE TO LEVERAGE MY LENS
AS A BLACK DISABLED WOMAN WHO
WAS BORN WIN A FORM OF MUSCULAR
DYSTROPHY AND ALSO DIDN'T ALWAYS
USE MOBILITY AIDS SUCH AS A CANE
AND MANUAL WHEELCHAIR AND I ALSO
USE A VENTILATOR TO ASSIST MY
BREATHING AND WHEN I'M RESTING
AT NIGHT.
THERE WAS GROWTH TAKING PLACE
AND A WONDERFUL JOURNEY IN
COALESCING WITH OTHER
LIKE-MINDED FOLKS IN
COLLABORATIVE EFFORT WORKING
TOWARDS BENEFICIAL CHANGE IN
WORKING ON ADVISORY BOARDS AND
THE DISABILITY POLICY CONSORTIUM
AND THE NATIONAL RESEARCH CENTER
FOR PARENTS WITH DISABILITIES
ADVISORY BOARD AND ALSO OPEN
DOOR ARTS.
I WAS ABLE TO GET TOGETHER WITH
PEOPLE FROM DIFFERENT WALKS OF
LIFE AND SHARING THEIR
EXPERIENCE BEHIND WHAT IT MEANS
TO LIVE WITH DISABILITIES
WHETHER THEY'RE A PARENT, NOT A
PARENT, CHRONIC ILLNESS, WHETHER
THEY ARE NONPROFITS OR PARTY
GOERS OR COMMUNITY BUILDERS AND
HAVE ALL THESE DIFFERENT LENSES
AND LIVED EXPERIENCES THAT
PROVED CRITICAL FOR GIVING INPUT
ON MANY QUALITY OF LIFE CONCERNS
AND AROUND HOUSING AND HEALTH
CARE, EDUCATION AND EMPLOYMENT
AND THEY IMPACT SO MUCH OF WHAT
WE DO ON A DAILY BASIS.
IT IMPACTS HOW YOU SHOP, DINE,
SOCIALIZE HOW YOU VOTE AND
PARENT AND OTHER AREAS THAT
IMPACT US IN SO MANY WAYS.
MY INPUT AND INVOLVEMENT IN
DISABILITY RIGHTS ADVOCACY COMES
AND BRINGS ALL THOSE IDENTITIES
I SPOKE TO ABOUT BEING A BLACK
DISABLED AND CAREGIVER AND DO SO
WITH THE HOPES OF HELPING TO
INFORM PUBLIC AND SOCIAL POLICY
AND IN A BENEFICIAL AND
MEANINGFUL WAY AND OPPORTUNITIES
COME FROM ACROSS THE LANDSCAPE
OR LACK OF MEANINGFUL DEPICTIONS
AND THERE'S A DANGER WITH THAT
WHEN WE'RE NOT WELL INFORMED
ABOUT WHEN IT MEANS TO LIVE WITH
A DISABILITY OR DISABILITIES.
BECAUSE THEY CAN MAKE THEIR WAY
INTO THE LEGISLATURE.
I VALUE THE CONVERSATIONS SUCH
AS WE'RE HAVING TODAY EVOLVING
AROUND WORKING WITH OTHER PEOPLE
ON PROFIT BOARDS AND HAVING
MEANINGFUL CONVERSATIONS AND
DIALOGUES TO HEAR OTHER PEOPLE'S
WAYS OF LIFE AND HOW THEY MOVE
SYSTEMS AND MAKING CONTRIBUTIONS
IN MANY LOCATION S LOCATIONS ANL
CONTRIBUTIONS THAT IMPACT THE
ENTIRE COMMUNITY.
SO WE WORK WITH SISTER
ORGANIZATIONS.
HERE IN BOSTON WE HAVE BOSTON
CENTER FOR INDEPENDENT LIVING
AND DISABILITY POLICY CONSORTIUM
AND WORKING ON THE MAYOR'S
COMMISSION WITH DISABLED PERSONS
AND THESE ARE PEOPLE FROM ACROSS
THE CITY AND STATE AND THERE'S
VALUE IN THAT BECAUSE WHEN WE
TALK ABOUT THESE IMPORTANT
CONVERSAT
CONVERSATIONS WE GET TO
UNDERSTAND PEOPLE IN A
COMPREHENSIVE WAY AND THAT'S
IMPORTANT WHETHER YOU WERE BORN
WITH A DISABILITY OR ACQUIRED IT
AND THAT'S THE IMPACT THAT I
THINK IS A WONDERFUL MISSION AND
JOURNEY AND JOB I HAVE IN
LENDING MY EXPERIENCES IN THAT
KIND OF WAY SO I'M HAPPY TO BE
HERE AND SHARE MY PERSPECTIVE,
OUR PERSONAL PERSPECTIVE IN
COALESCING WITH OTHER PEOPLE SO
THANK YOU VERY MUCH FOR HAVING
ME HERE TODAY.
>> THANK YOU FOR SHARING YOUR
STORY.
NEXT WE HAVE INDU NAVAR.
>> THANK YOU FOR HAVING ME.
I'M HONORED TO SHARE OUR STORY.
EVERYTHING IS FOCUSSED ON FUTURE
OF DIGITAL BIOMARKERS, BRINGING
IN TECHNOLOGY, INNOVATIONS TO
NEUROLOGICAL DISEASES STARTING
WITH ALS AND YOU CAN SEE SOME OF
THE PARTNERSHIP IN THE
PRESENTATION.
WHY IS THIS IMPORTANT?
MOST ARE HERE BECAUSE IT'S A
GROWING PROBLEM.
IT'S NOT A PROBLEM CONTAINED AND
THIS IS MY INTEREST IN LOOKING
AT THIS MORE DEEPLY AND THE COST
OF NEUROLOGICAL DISEASES IS
EXPENSIVE AND I WILL SHOCK WITH
MY OWN JOURNEY I'LL TALK A
LITTLE BIT ABOUT GOING THROUGH
MY HUSBAND'S DIAGNOSIS OF ALS
AND SEEING HOW THE BIG PHARMA
COMPANIES ARE MOVING AWAY FROM
NEUROLOGICAL DISEASES BECAUSE
THERE'S NO WAY TO MEASURE IN AN
OBJECTIVE WAY IN A TRIAL AND
THIS MONTH IS MY HUSBAND'S
BIRTHDAY MONTH AND WE LOST HIM
IN 2019.
MY HUSBAND WAS BE AN INNOVATOR
AND A MEMBER OF CLOUD, AMAZON
AND WAS THERE 18 YEARS AND BUILT
TOOLS USED BY MANY RESEARCHERS
TODAY AND THE SHOCK WAS WE WENT
EARLY IN THE NEUROLOGIST AND
WHAT WE HEARD WAS COME BACK
EIGHT, NINE MONTHS AND SEE HOW
MUCH WORSE YOU'RE GOING TO BE
AND STILL RINGS IN MY EAR ABOUT
WASTING AWAY THE TIME WHEN HE'S
SUPPOSED TO BE WAITING TO GET
BETTER AND WAITING TO BE
DIAGNOSED.
AFTER HE PASSED AWAY ONE THING
MY FATHER TOLD ME WAS NOW THAT
YOU KNOW SO MUCH ABOUT THE
DISEASE AND SORRY, I GET TEARY
EYED WHEN I TALK ABOUT THE
STORY, WHAT ARE YOU GOING TO DO
WITH IT IS THE QUESTION?
WHEN I LOOKED AT HARD AT WHAT IT
IS, I'M NOT AN NEURO SCIENTIST
OR M.D. BUT HAVE A LOT OF
INFORMATION AND EXPERIENCE THAT
I CAN BRING TO THE TABLE AND I'M
AN ENTREPRENEUR.
I BUILT A LOT OF SOFTWARE
COMPANIES INCLUDING WHAT TODAY'S
WEB MD AN ORIGINAL TEAM MEMBERS
AND WORKING IN THIS COMMUNITY
BACK AFTER HAVING LOST MY
HUSBAND I HAD TO THINK HOW DO I
FACE SMIS DISEASE I NEEDED TO
LOOK AT MY FEARS AND MY HUSBAND
INSPIRES ME EVERY DAY TO DO
THAT.
IN HIS HONOR I WANT TO IMPACT
THE DISEASE BECAUSE I DON'T WANT
HIM TO BE REMEMBERED AS SOMEONE
WHO LOST THEIR LIFE TO ALS BUT
BRING TREATMENT AND BIOMARKERS
TO THIS DISEASE.
THE SKILL SET I HAVE IS LIKE
JANET MENTIONED I STARTED MANY
COMPANIES AND SIT ON A RESULT
BOARDS.
I DO HAVE ENDURANCE AND KNOW HOW
TO BUILD ALIGNMENT AND FAST
LEARNER AND EXPERIENCE IN
BUILDING TEAMS AND GROWING A
BUSINESS.
WITH THESE TRAITS AND SKILLS I
SET OUT TO START EVERYTHING ALS
AND FOUNDATION AND THE PRIMARY
GOAL IS TO LOOK AT AT THE
SUBJECTIVE MEASURE OF
PROGRESSION TRACKING.
AND I HAD NO IDEA IT WAS THE
GOLD MEASURE FOR PROGRESSION
TRACKING.
MY HUSBAND WOULD LOOK TO ME FOR
THESE QUESTIONS AND I WOULD
ANSWER AND I THOUGHT THEY WERE
AT&T, MOBILE SURVEY QUESTIONS
HOW DO YOU RATE IT 0 TO 4?
I KNOW WHAT I ZERO IS BUT NOT A
TWO OR THREE.
AND LOOKING AT THE PLACES TO
IMPACT WITH OUR BACKGROUND WAS
TO LOOK AT CLINICAL END POINTS
AND DIAGNOSIS.
WE STARTED AS ALS IS A TRICKY
DISEASE IT TAKES 12 TO 18 MONTHS
TO GET DIAGNOSED.
IN MY HUSBAND'S CASE MORE THAN
TWO YEARS AND THE SURVIVAL
AVERAGE IS TWO TO FIVE YEARS.
THERE'S OPPOSING FACTORS IN WHEN
WE DIAGNOSE AND NOT HAVING
DEFINITIVE DIGITAL BIOMARKERS.
WE TEAMED UP WITH PEOPLE WHO ARE
DOMAIN EXPERTS AND MOVE WITH
THIS ON A DAY TO DAY BASIS.
IT'S NOT A BOARD WHERE WE TOUCH
BASE ONCE IN A WHILE AND THEY
WORK WITH US AND JAMES AND
AMAZING DIGITAL BIOMARKER
KNOWLEDGE FROM MGH AND A
NEUROLOGIST AT JOHNS HOPKINS AND
AN A.I. EXPERT AND MTH WITH A
DATA PLATFORM.
THESE ARE OUR CORE GROUP OF
ADVISORS AND WE STARTED OFF WITH
BUILD PATIENT COMMUNITY AND LIKE
WALTER SAID HOW DO WE GET THE
TRUST WITHIN THE PATIENT
COMMUNITY TO BECOME A RESEARCH
PARTNER AND THAT'S WHERE WE
EXPERIMENTED AND WERE SUCCESSFUL
IN BUILDING, PATIENTS ARE NOT
EXTERNAL ENTITY.
THEY NEED TO BE IN THE RESEARCH
THAT ARE RESEARCH PARTNERS AND
THEN DO DATA COLLECTION AND HAVE
A DATA SCIENCE TEAM WITH A.I.
PARTNERS.
WE DO DATA QUANTIFYING
PROGRESSION AND PREDICTING THE
DISEASE.
EVERYTHING ALS AS A DASHBOARD DO
PATIENT-MEDIATED DATA COLLECTION
AND STARTED WITH LOOKING AT
SPEECH AND VIDEO AND KICKED OFF
A PROJECT ON BREEDING COGNITION,
GROSS MOTOR SENSOR AND SPEECH.
IT'S ALL OPEN INNOVATION AND
SCIENCE.
THIS IS AN IRB APPROVED PROTOCOL
WE DO AND WE ENGAGE WITH
START-UPS WHO HELP WITH THE DATA
AND WE EMBED THESE AS
EXPERIMENTAL BIOMARKERS IN PHASE
1 TO 2 TRIALS AND GET IT TO WORK
DIRECTLY WITH FDA.
WE HAD A LOT OF CONVERSATIONS.
WE HAVE AN M.O.U. WITH CRITICAL
PATH INSTITUTE AND WORKING WITH
THE LAW FIRM TO TAKE OUR
FINDINGS AT GET THE FDA APPROVED
TO BE A DIGITAL BIOMARKER.
IT'S BRINGING STAKEHOLDERS
TOGETHER AND WHY THIS IS
IMPORTANT IS THAT THE WAY WE ARE
DOING THE OPEN INNOVATION IS
TRADITIONALLY THE RESEARCH IS
DONE FROM AN EXTERNAL
PERSPECTIVE AND STUDIES ARE DONE
WITH SMALL COHORTS AND SMALL
FINDINGS AND NOW WE'RE OPENING
IT UP TO MAKING IT AN OPEN DATA
PLATFORM ANALYZED BY MANY
RESEARCHERS AT THE SAME TIME AND
TOLD THE CURRENT MODEL IS YOU
BUILD EACH NEW STADIUM FOR EACH
GAME AND WHAT WE'RE TRYING TO DO
IS TRYING TO PLAY A SIMULTANEOUS
GAME IN THE SAME STADIUM BUILT.
JUST AS QUICK METRICS, OUR
PATIENT COMMUNITY HAS BEEN
GROWING.
WE HAVE OVER 5,000 PEOPLE WHO
ARE A CORE PART OF OUR
COMMUNITY.
BOTH PATIENTS AND THE CAREGIVERS
AND WE HOST EVENTS EVERY TWO
WEEKS WE CALL ALS TALK SERIES
AND BRING IN WHO'S-WHO AND THE
RESEARCHERS TO COME IN AND TALK
TO THE PATIENT COMMUNITY.
ALL OUR RESEARCH IS 100% REMOTE.
WE'RE SET UP AS A VIRTUAL GROUP
AND IT'S REALLY THE FEELING OF A
PATIENT FROM PATIENT'S LIVING
ROOM TO THE RESEARCHERS LIVING
ROOM ASKING ANY QUESTIONS.
THIS IS VERY WILDLY SUCCESS FFU
PROGRAM AND WE HAVE VISITORS WHO
HAVE CONSUMED 160 HOURS OF
CONTENT.
AND FOR THE PEOPLE LIVING WITH
ALSO TO BE PART OF THE RESEARCH.
AND WITH THAT WE RECRUIT FROM
SOCIAL MEDIA AND OUR COMMUNITY
AND RESEARCH AND THE FIRST WE
KICKED OFF WAS WITH
MODALITY.A.I. WE DID FACIAL
VIDEO AND SPEECH AND COGNITION
AND TELL YOU ABOUT THE METRICS
IN THE NEXT SLIDE.
THE SEARCH ENGINE INTERACTIONS
THAT ARE FINE MOTOR STUDY BY
MICRORESEARCH AND WE LOOKED AT
THE DATA WITH HOW THE FINGER IS
MOVING AND TYPING AND MOUSE
MOVEMENT AND SPELL CHECKER AND
LOOKED AT WHEN WOULD IMPACT ALS
PATIENTS IN CONTROLS AND THERE
WAS A CLEAR DISTINCTION FOR
PEOPLE WHO GOT DIAGNOSED WITH
ALS VERSUS CONTROLS.
THE NEXT STUDY WE JUST KICKED
OFF WAS WITH A START UP WHERE
THE SENSORS GO TO THE SHOES AND
WE'LL BE ABLE TO LOOK AT GAIT,
STRIDE, BALANCE LONG WITH
BREATHING WITH ANOTHER COMPANY.
OUR GOAL IS TO AUTOMATE THE FOUR
COMPONENTS OF THE ALS SCORE AND
WE'VE BEEN GOING AT IT AND
ATTEMPTED TO DO ALL OF THEM THIS
YEAR.
SO THE NUMBERS IN 14 MONTHS WE
WERE ABLE FOR OUR FIRST STUDY WE
GOT OVER 1,000 HOURS OF DATA.
THIS IS THE PATIENTS AND
PARTICIPANTS WOULD SPEND ABOUT
10 MINUTES EVERY WEEK AND THIS
WAS DIFFERENT QUESTIONS AND THE
PARTICIPANTS WOULD RESPONSE AND
THIS WAS FOR SPEECH COLLECTION
AND WE COVERED GEOGRAPHY
DISTRIBUTION EXCEPT MONTANA WAS
THE LAST STATE HARD TO GET AND
WE GOT ABOUT 750 PARTICIPANTS
WHO CONSENTED IN THE 14 MONTHS
TO BE IN OUR STUDY.
ONE THING WE TALKED ABOUT IS
ETHNIC DIVERSITY.
THIS IS VERY IMPORTANT TO US.
WE LOOKED AT THE U.S. POPULATION
AND OUR STUDY AND WE HAVE
AMERICAN INDIAN POPULATION OR
NATIVE HAWAIIAN POPULATION AND
HAVE TO DO BETTER IN SOME OTHER
CATEGORIES BUT WE DO PAY CLOSE
ATTENTION TO THE ETHNIC
DIVERSITY AS WELL AND
SUBPOPULATION DIVERSITY AS YOU
CAN SEE.
WE ARE TRACKING WELL WITH ASIAN
SUBPOPULATION DIVERSITY.
OUR GOAL IS TO DO THE MULTI-MODE
DIGITAL BIOMEDICAL AND GO
THROUGH FINE MOTOR, GROSS MOTOR
AND RESPIRATORY AND THE TIME
LINE BY MID NEXT YEAR WE'D HAVE
DATA FROM ALL THE BIG FOUR
CATEGORIES.
WE DO HAVE SOME PUBLICATIONS YOU
CAN GO TO OUR WEBSITE EVERYTHING
ALS DOT ORG AND LOOK AT THE
PUBLICATION.
WE'RE BRINGING IN A LOT OF
PHARMA COMPANIES TO WORK WITH US
AND SOME STAKEHOLDERS WHY
THEY'RE PARTNERING WITH US IS
BECAUSE THEY WANT TO SEE THE
SPEED AND EXECUTION.
THIS IS A PLATFORM THAT WE ARE
BUILDING WITH ALS AS OUR FIRST
FOCUS BUT IT'S APPLICABLE FOR
MANY OTHER DISEASES AND WE WANT
TO BE AN OPEN INNOVATION
PLATFORM AND MAKE IT AVAILABLE
FOR ALL OTHER DISEASES.
THANK YOU VERY MUCH.
>> THANK YOU VERY MUCH FOR
SHARING YOUR EXPERTISE AND YOUR
LEARNINGS WITH THE ENTIRE
NEUROLOGICAL COMMUNITY.
YOU'LL HAVE TO EXPRESS OUR
APPRECIATION TO YOUR FATHER FOR
ASKING THAT IMPORTANT QUESTION.
WE HAVE A QUESTION AS WE BRING
UP THE REST OF OUR PANEL I THINK
WE'LL GIVE TO YOU.
IF YOU'RE WORKING PREDOMINANTLY
ONLINE TO COLLECT DATA WITH A
WEBSITE AND SOCIAL MEDIA AS YOUR
FORMS OF OUTREACH, HOW CAN YOU
REACH A REPRESENTATIVE
POPULATION.
DO YOU HAVE THOUGHTS ON THAT?
>> THIS IS THE BEST WAY.
PATIENT ADVOCACY GROUPS HAVE TO
GET MORE INVOLVED IN RESEARCH
LIKE THIS BECAUSE WE WERE ABLE
TO BRING IN DIVERSE POPULATION
BECAUSE OF THE WHOLE SOCIAL
MEDIA AND WEBSITE RECRUITING
BECAUSE WHEN TRADITIONALLY THE
RESEARCH IS DONE BY
INSTITUTIONS, YOU REALLY GET
ONLY THE POPULATION AROUND THAT
INSTITUTION WHO GO TO THAT
INSTITUTION AND NOW WE'RE ABLE
TO FIND AND ONE IS REMOTE AND
SECOND IS BECAUSE PEOPLE IN THE
REMOTE PLACES, WANT TO BE PART
OF THE COMMUNITIES SO THEY DO
ACTIVELY ENGAGE IN SOCIAL MEDIA
AND SOMETIMES WE ARE A B TO C
COMPANY WHERE WE SEEK OUT
COMMUNITIES WHERE THEY ARE AND
BRING THEM IN AND USE A LOT OF
APPROXIMATE TO ATTRACT DIFFERENT
COMMUNITY WE NEED TO ENGAGE IN
OUR RESEARCH.
I HOPE THAT ANSWERED THE
QUESTION.
>> AND YOU'RE ABLE TOE TRACK
THAT AS WELL.
YOU KNOW YOU'RE REACHING ALL
POPULATIONS FOR THE ALS
COMMUNITY?
AND WE HAVE TO FIND WHERE ARE
THE PEOPLE.
CERTAIN COMMUNITIES WERE NOT IN
THE MAINSTREAM LIKE MY HUSBAND
WOULDN'T BE IN FACEBOOK.
WE ARE TRYING TO MAKE SURE WE
CAN REACH AND DO MORE PR WHY THE
STUDY'S IMPORTANT AND WE'RE ON
TVs AND SOME NEWS CHANNELS AND
COMMUNITIES.
WE TAKE THE ALS PATIENTS RE-MOTE
COMMUNITIES AND HIGHLIGHT THEIR
STORY WITH OUR
PR EFFORT.
WE HAD PEOPLE WHO CAME TO OUR
WEBSITE AND WANT TO BE INVOLVED.
>> HAVING THE PATIENT
REPRESENTATIVE RECORD THE ENTIRE
RESEARCH INVOLVEMENT FROM HER
PERSPECTIVE I THINK WAS
BRILLIANT AND HOPEFULLY
CONTRIBUTED SIGNIFICANTLY TO THE
EASE OF RECRUITMENT.
>> THANK YOU.
IT'S SOMETHING I CONTINUE TO DO
MOVING FORWARD.
I AGREE THAT SOMETIMES NO MATTER
HOW HARD A RESEARCHER OR
CLINICIAN TRIES TO EXPLAIN
THINGS IN PATIENT-FRIENDLY TERMS
WHO DOES BETTER THAN THE PATIENT
THEMSELVES.
>> WE HAVE A QUESTION TO THE
PANEL IF THEY THINK VOLUNTEER
PARTICIPATION CAN MAKE A
DIFFERENCE IN THE CLIMATE OF
PATIENT ENGAGEMENT.
>> I AGREE.
DESPITE THE FACT I'M USING
PATIENT ENGAGEMENT, I DON'T USE
THE WORD PATIENT IN THE PAPERS I
WRITE OR WHAT I REFER TO AS
PATIENTS SO PATIENT SUBJECT IS
NOT THE BEST TERM BUT I RERCH TO
OUR PATIENT PARTNERS TO WHAT
THEY THINK ABOUT THE TERMINOLOGY
AND WHAT WOULD BE BETTER.
>> WE TRIED TO REACH OUT TO
PEOPLE WITH ALS IT'S MORE HOW WE
TREAT THEM.
FOR MY, MY HUSBAND WE WERE SMART
PEOPLE AND HEARD FROM
NEUROLOGISTS OVER AND OVER AGAIN
I'VE NEVER SEEN SO MUCH DATA
BEING COLLECTED WE HAD A DATA
SCIENCE TEAM AND WE LITERALLY
HAVE SO MUCH ABOUT HIS DATA
BECAUSE WHEN THE NEUROLOGIST
COME BACK AFTER EIGHT MONTHS I'M
LIKE WHAT WILL HAPPEN IN EIGHT
MONTHS.
I CAN TELL YOU IN WEEKS HOW MANY
MUSCLES ARE DEGRADING WITH
DIFFERENT SENSORS AND I DON'T
NEED TO COME TO THE FACILITY FOR
YOU TO MOVE HIS ARMS AND LEGS.
WE COLLECTED A LOT OF THE DATA
BUT WE WERE STILL NOT INTEGRATED
BECAUSE THE SYSTEM WAS NOT SET
UP TO BE INTEGRATED.
AND WE SET UP FEEDBACK MEETINGS
AND KICKED OFF A STUDY WITH
LARGE ORGANIZATIONS AND WE HAVE
ABOUT SIX SUPER USERS TO COME
IN, PATIENTS OR PEOPLE LIVING
WITH ALS TO GIVE FEEDBACK.
WE CALL IT A BETA LAUNCH.
WE'RE BRINGING IN ALL THE
SOFTWARE TERMS.
IT'S A BETA LAUNCH AND NEED
FEEDBACK FROM PEOPLE SAYING HOW
IS IT, IS THERE SOMETHING WE
NEED TO SPEAK IN THE PROTOCOL
AND ASKING THEM TO GIVE FEEDBACK
ON HOW IT WOULD BE.
AND WE WERE NOT REALLY
LEVERAGED.
>> WHEN SO MUCH CONFIDENTIALITY
IS DEIDENTIFIED IN PAPERS AND
KEEPING THAT FOCUS CENTRALIZED.
THERE'S AN IMPORTANT INCENTIVE
WORKING WITH DIFFERENT PEOPLES
AND BACKGROUNDS.
IT'S EASY TO LOSE SIGHT.
>> THAT'S WHERE WE HAVE A CLEAR
DISTINCTION OF IRB AND IT GOES
TO DE-IDENTIFIED.
IT'S LIKE PEOPLE ASKING ME WHAT
SHOULD I DO?
WE RECOGNIZED THE FRICTIONS AND
TRIED TO REMOVE THE FRICTION.
>> WE NEED A MODERATOR.
>> I HAVE QUESTIONS ARE YOU
INCLUDING PATIENTS WITH LIMITED
ENGLISH PROFICIENCY?
YES, FOR ENGLISH AND UNITED
STATES.
WE'RE A YOUNG ORGANIZATION.
WE STARTED YEARS AGO AND WE'RE
NOW THINKING ABOUT SPANISH
SPEAKING AND ALSO EXPANDING SO
IT'S IN THE WORKS.
>> I MENTIONED WE WERE USING THE
TERM MEDICATION OVERUSE HEADACHE
AND THIS IS THE OFFICIAL TERM.
THERE'S PUBLISHED DIAGNOSTIC
CRITERIA AND THAT'S WHAT IT'S
CALLED.
AND IT DIDN'T SIT WELL BECAUSE
FOR SOME IT HAS THE CONNOTATION
YOU'RE BLAMING THE PATIENT FOR
THEIR OWN CONDITION.
IT REFERS TO THE VAST MAJORITY
OF PEOPLE IF THEY'RE HAVING A
MIGRAINE THEY'LL WANT TO TREAT
IT TO REDUCE THEIR SYMPTOMS.
AND USING MEDICATIONS CAN RESULT
IN CAUSING MORE FREQUENT
HEADACHES.
THERE'S BEEN SEVERAL YEARS
DEBATE AND TERMINOLOGY TO PUT
LESS BLAME ON THE PATIENT.
WE WERE STUCK BECAUSE AND THE
WORD MEDICATION OVERUSE IN IT
AND THERE WAS AN ATTORNEY
GENERAL TRIAL.
WE MADE A POINT OF ASKING
INVESTIGATORS AND COORDINATORS
TO TAKE A MINUTE OR 30 SECONDS
AND WALK THE POTENTIAL
PARTICIPANTS THROUGH THE
TERMINOLOGY AND WHY WE'RE DOING
IT.
MAKING SOMEONE UNDERSTAND HOW
YOU ARE USING THE TERMINOLOGY
PROBABLY ALL YOU CAN DO UNTIL
YOU ADD IT TOGETHER.
I HOPE THIS DOESN'T SOUND
CONTROVERSIAL BUT I SPEND TIME
WITH FAMILIES AND PATIENTS I
WORK WITH AND IT TAKES AWAY FROM
THE BIGGER ISSUE.
AND THESE ARE CREATING SO MANY
CONTROVERSIES WHEN THEY
SHOULDN'T.
NO ONE IS QUESTIONS THE -- WE
NEED TO LOOK AT THE PATIENT
EXPERIENCE.
>> AND THE WORD SUBJECT, I'M NOT
A BIG FAN OF SUBJECTS.
>> MY APOLOGIES.
MY SCREEN WENT OUT.
WOULD YOU TALK ABOUT WORKING
WITH FAMILIES AN CHILDREN
BECAUSE THEY'LL BENEFIT FROM THE
PROGRAM.
YOU CAN'T GO TO THEM DIRECTLY.
CAN YOU TALK ABOUT THAT?
YOU HAD QUESTIONS AS WELL.
>> I WORK WITH CHILDREN AND
ADULTS TO HAVE THE CAPACITY TO
CONS
CONSENT I THINK FAMILIES ARE
CENTRAL TO THE CARE.
AND ANYONE WHO HAS A CHRONIC
CONDITION REQUIRES THE FAMILY
STRONGLY.
THEY'RE PART OF THIS WHEN YOU'RE
WORKING WITH THE COMMITTEE
THAT'S WHEN YOU THINK CHILDREN
CAN PRESENT WE LOOKED AT THINGS
THAT MAY BE COMPLICATED TO
UNDERSTAND BUT WE REACH THE
FAMILIES AND WITH THE FAMILIES
IN PERU THE OLDER ADOLESCENTS WE
HAD A COUPLE OF STUDENTS THAT
WERE HELPING ORGANIZE THE STUDY.
>> I WANT TO SAY IN SO MANY
COMMUNITIES OF COLOR WE LIVE
INTERDEPENDENTLY AND MAY BE
SANDWICHED BETWEEN TAKING CARE
OF OURSELVES, OUR CHILDREN AND
ELDER AUNT OR PARENT.
SOMETIMES WHEN WE TALK ABOUT
RESEARCH AND EVEN IN CLINIC
SETTINGS WHEN YOU GO TO THE
DOCTOR'S OFFICE AND HAVE STAFF
SAY, OH, YOU BROUGHT THE WHOLE
FAMILY.
THERE'S A SUBTEXT THERE THAT'S
NOT SO WELCOMING.
WE NEED TO BE MINDFUL OF THINGS
LIKE THAT ARE OFF PUTTING MAKING
FAMILIES AND PATIENTS NOT FEEL
INCLUDED BUT CONTRIBUTE TO
HEALTH CARE DISPARITIES.
I WANTED TO SHARE THAT QUICKLY.
>> WE STARTED IN MAY OF 2020 AND
THE PROJECT WAS THE FIRST
RESEARCH TO MARCH 2020 AND I
DON'T KNOW WHAT A PRE-COVID
WORLD LOOKED LIKE BUT WE'RE THE
POST-COVID.
MOST PEOPLE HAD AN iPAD OR 4G OR
3G IN THEIR PHONE.
WE DON'T REQUIRE A LOT OF
BANDWIDTH.
IT'S ALL THEY NEED IS A PHONE OR
iPAD.
AND WE KNOW WITH ALS PATIENTS IS
MORE THAN 50% OF THE PEOPLE LIVE
50 TO 75 MILES FROM THEIR
NEUROLOGY CENTER.
ASKING THEM TO COME TO THE
CENTER TO DO THE STUDY, THAT IS
NOT A VERY GOOD WAY OF
COLLECTING DATA OR TRYING TO DO
THIS RESEARCH AND SECOND LY
BECAUSE OF THE DETERIORATION IN
ALS PATIENTS THERE'S A LOT OF
FALLOUT AND WE WERE ABLE TO
RETAIN A LOT OF THE PEOPLE
BECAUSE PEOPLE COULD PARTICIPATE
REMOTELY AND WANTED TO MAKE SURE
WE HAVE BANDWIDTH AND USABLE BY
PEOPLE WHO HAVE LIMITED WI-FI
NETWORKS.
IT DOESN'T ALWAYS HAVE TO BE
CONNECTED AND THINGS LIKE THAT.
WE DO PAY A LOT OF ATTENTION TO
THOSE DETAILS.
I MOST PEOPLE SHOULD HAVE A 4G
OR 3G CONNECTION.
>> ONE OF THE SPACES WE TALK
ABOUT IN THE GOVERNMENT ARE
ABOUT EDUCATING US AND BEING
RESPECTFUL TO THE PEOPLE
AFFECTED BY THE DIFFERENT
NEUROLOGICAL DISORDERS AND
HELPING OUR RESEARCH CLINICIANS
UNDERSTAND THAT TOO.
AND TRYING TO CONNECT DOTS IN
TERMS OF DIFFERENT EDUCATIONAL
MATERIALS WE PUT OUT HAVING THEM
SO THE WHOLE SYSTEM ENDS UP KIND
OF GROWING TOGETHER AS WE ALL
COME TO REALIZE THE DISTINCTIONS
AND SO I'M CURIOUS ABOUT HOW ALL
OF YOU PERCEIVE THE DIFFERENCES
IN THE TYPES OF INFORMATION
DIFFERENT GROUPS NEED ACROSS THE
PIPELINE, IF YOU WILL, AND THE
DIFFERENT PLACES PEOPLE MIGHT
LOOK FOR THOSE MATERIALS.
>> I CAN START BY COMMENTING
CERTAINLY WHEN I LEARNED HOW TO
DO RESEARCH AND BACK A COUPLE
DECADES AGO, INCLUSION OF
PATIENTS AS EQUITABLE PARTNERS
WAS NOT PART OF THAT.
AND HOW TO BEST INCORPORATE
PATIENTS AS RESEARCH PARTNERS.
IT HAS TO BE AND GIVING EXAMPLES
HOW TO BEST ENGAGE PATIENT
PARTNERS.
>> THERE'S SILENCE WORK THAT
GOES BEHIND THE SCENES.
TRADITIONALLY THAT HAS NOT BEEN
RECOGNIZED AND THOSE SILENT
HOURS OF WORK HAVE A COST AND
WHEN I SAID WE HAVE GRANTS TO
LOOK AT GENOMICS IN DISABILITIES
AND THERE'S A MULTI-NATIONAL
GRANT IN LATIN AMERICA AND HAVE
PARTNERS IN THE UNITED STATES
AND DO STUDIES LOOKING AT
SYSTEMS AND THE SALARIES OF
PEOPLE AND SETTING THE STUDY WAS
MORE IMPORTANT BECAUSE
INFRASTRUCTURE DOESN'T EXIST.
THE TRUTH IS IT'S NOT
SUSTAINABLE UNLESS IT'S
RECOGNIZED.
AND RECOGNIZE THE WORK AND
EVERYBODY THAT'S PART OF IT.
>> WE NEED TO BE REACHING OUT TO
THE VERY FOLKS THAT WE ARE
INCLUDING.
AND USING PEER RESEARCHERS TO
INFORM YOUR PRACTICE THE PEOPLE
YOU ARE HELPING OR ADVOCATING
FOR ARE INCLUDED IN THE
CONVERSATIONS BECAUSE THEY CAN
HAVE IT BE A SECONDARY SOURCE.
GO TO THE PRIMARY SOURCE.
I LEARN ALL THE TIME AND IT'S
COME TO BETTER INFORM ME.
AND THERE'S GROUPS LIKE FACEBOOK
AND TWITTER AND DISABILITY
TWITTER.
VERY INFORMATIVE.
AND PEOPLE ARE SHARING THEIR
HISTORY AND LIVED EXPERIENCE
THROUGH BLOGS AND WRITTEN WORD
TYPICALLY BECAUSE THEY DON'T GET
THAT CONTROL ACROSS THE MEDIA
LANDSCAPE.
WE HAVE TO HAVE THEM HEAR US AND
THAT MAY SHOW UP IN RESEARCH AND
OUTREACH.
I WANT TO INCLUDE THOSE POINTS.
>> DO YOU HAVE ANOTHER THOUGHT
TO ADD ALONG THOSE LINES?
>> I FEEL ONE THING ABOUT
REACHING OUT TO PEOPLE THROUGH
SOCIAL MEDIA AND DIFFERENT
ETHNIC DIVERSITY AND WHAT I
REALIZED IS PUTTING IT OUT AND
SAYING COME AND JOIN US IS NOT
ENOUGH.
WE NEED TO SET UP A PLATFORM
WHICH HAS A CONTINUOUS
ENGAGEMENT AND WHEN WE SET UP
THIS EVERY TWO WEEKS RESEARCHERS
ARE COMING TO YOU AND THAT'S A
BIG DRAW BECAUSE FOR US TO GO
GET A NEUROLOGY APPOINTMENT FROM
A DIFFERENT -- IT'S LIKE NOT
FEASIBLE.
THIS WAY SOMETIMES 500 LIVE
PEOPLE ATTEND BECAUSE THEY WANT
ACCESS TO WHAT THAT RESEARCHER
OR NEUROLOGIST OR PHARMA
COMPANIES SAY.
THIS HAS BEEN ALMOST TWO YEARS
AND PEOPLE CALL IT HAPPY HOUR.
AND THEY BUILT THIS ON BASED HOW
AS AN ENTREPRENEUR I WOULD GO TO
THIS EVENT AND THEN MEET
LIKE-MINDED PEOPLE AND SAY WHAT
ARE YOU DOING AND WHAT DOES YOUR
PITCH LOOK LIKE?
IT'S THE SAME.
WHEN YOU START GETTING
DIAGNOSED, YOU HAVE NO IDEA, I
DIDN'T EVEN KNOW WHAT ALS WAS.
YOU HAVE TO SOME BASIC QUESTION
AND I'D LOVE TO MEET OTHER
PEOPLE WHO ANSWERED THE
QUESTIONS.
AFTER THE EVENT PEOPLE HAVE
ABOUT 30, 45 MINUTES AND PEOPLE
DON'T LEAVE.
SOMETIMES WE HAVE TO GO, OKAY,
IN TWO WEEKS WE'LL MEET.
IT'S REALLY A SET UP LIKE PEOPLE
COME IN AND THEY NOW RECOGNIZE
EACH OTHER AND CAN FEEL
COMFORTABLE.
IT TOOK TIME.
IN THE BEGINNING WHEN WE SET IT
UP WE TOLD PEOPLE TO COME IN AND
SHOWED THEM HOW TO TURN OFF
THEIR CAMERA AND MICROPHONE IF
THEY DON'T WANT TO BE SEEN AND
PARTICIPATE AND BUILDING TRUST
IN THE COMMUNITY TAKES TIME AND
IT'S RINSE AND REPEAT AND WHAT
HAS WORKED WELL AND KEEP DOING
IT AND PEOPLE WILL START FEELING
COMFORTABLE AND THAT'S BEEN MY
EXPERIENCE BUILDING AN
ENGAGEMENT PLATFORM WHERE PEOPLE
WILL COME TOGETHER.
WE'RE COVID MAYBE AND PEOPLE
WANTED TO CONNECT.
HAVING SOMETHING ONLINE IS
IMPORTANT TO SET UP AN ACTIVE
ENGAGEMENT AND HAVE THESE
DIALOGUES ABOUT WHAT'S GOING ON
AND HOW DO YOU CONTRIBUTE.
IT'S MAKING THAT PART OF THE
VALUE ADD AND PEOPLE WITH THE
DISEASE PART OF THE VALUE ADD.
>> I APPRECIATE THAT IMPACT OF
COVID AND THE TINY SQUARES ON
TEAM AND THOSE WHO CAN BE
REACHED AND THE MESSAGE AND
LEARNING MORE FROM THE
COMMUNITY.
>> THAT'S ALSO WHERE THE
DISABILITY COMMUNITY HAS BEEN
WE'VE BEEN ONLINE AND BEEN
NEEDING OPTIONS.
WE HAVE BEEN ASKING FOR
ACCOMMODATIONS FOR EONS AND IN
SO MANY SETTINGS AND PEOPLE SAY
NO.
THERE'S A LOT OF ABLISM.
PARTICIPATION SHOULD NEVER GO
AWAY AND YOU CAN SHUT OUT A WIDE
VARIETY OF PEOPLE WHO NEED THAT,
LOW INCOME, POOR FOLKS, PEOPLE
WHERE THEY DON'T HAVE WI-FI
ACCESSIBILITY.
THERE'S RELIGIOUS SETTINGS,
HOMELESS SHELTERS AND JUST
ANOTHER THOUGHT.
>> I SEE EVERYONE SHAKING THEIR
HEADS IN AGREEMENT.
THAT'S GOOD.
WE ARE LOOKING AT THE TIME AND I
REALIZE WE HAVE HIT THE END OF
OUR SESSION.
I WANT TO THANK ALL OF YOU.
JANET EXTENDS HER THANKS AND IS
SO SORRY BUT SHE KEPT FALLING
OFF ZOOM AND GRATEFUL FOR THE
OPPORTUNITY TO SPEAK WITH YOU
GUYS WHEN YOU WERE PLANNING THIS
TO COME IN AND FOR YOUR NIRNL
PRESENTATIONS.
I WANT TO THANK YOU SWELL --
THANK YOU AS WELL FOR YOUR
PRESENTATIONS.
BEFORE I GO INTO HOUSEKEEPING
I'M GOING TO ASK OUR DIRECTOR,
DR. KOROSHETZ TO MAKE A FEW
FINAL REMARKS FOR THE PUBLIC
SESSION.
>> THANK YOU.
THAT WAS A FASCINATING AND
IMPORTANT DISCUSSION.
YOU CAN'T DO THIS QUICK.
YOU HAVE TO BUILD THE
INFRASTRUCTURE.
YOU CAN'T HELICOPTER IN AND OUT
TO GET SUCCESS IN THE SPACE.
YOU'RE WORKING UPHILL BECAUSE
THE HEALTH CARE SYSTEM IS NOT
EQUITABLE.
AND YOU NEED TO DO IT TO BE
SUCCESSFUL AND OUTREACH TO
PEOPLE IRREGARDLESS OF THEIR
SOCIO ECONOMIC STATUS TO GET THE
WORK DONE FOR THEM I DON'T THINK
YOU CAN UNDERSTAND THE
COMPLEXITIES VERY QUICKLY.
IT TAKES TIME FOR PEOPLE TO GET
A SENSE OF WHAT THE DISEASES ARE
ABOUT.
AND THE EXPECTATIONS GOING INTO
THESE.
IT TAKES TIME IT MAY TAKE TIME
IN OUR SYSTEM.
I THINK THIS IS THE LAST TIME I
GET TO TALK TO YOU FOLKS AND
YOU'RE GOING INTO FEEDBACK
BREAKOUT SESSIONS NEXT RUN BY
PAUL GROSS AND THE NEXT PART OF
THE NON-PROFIT FORUM IS ALL
ABOUT NETWORKING AMONG THE
NONPROFIT AND THE NIH PEOPLE ARE
NOT ALLOWED TO BE THERE BUT IT'S
IMPORTANT TO GET A LOT OUT OF
HEARING FROM EACH OTHER IN THE
NETWORK SESSIONS.
AND THANKS TO THOSE WHO WORK
HARD IN PUTTING THIS TOGETHER.
EVERY YEAR WE WERE CHALLENGED BY
SAYING IT WAS GREAT LAST YEAR SO
WE CAN'T SCREW UP THE NEXT YEAR.
IT'S GETTING HARDER AND HARDER.
THANK GOODNESS I THINK WE DID IT
AGAIN.
AND PEOPLE INTERESTED IN THE
GROUP GOING FORWARD FOR NEXT
YEAR'S EVENT PLEASE LET US KNOW.
WE'RE ALWAYS LOOKING FOR MORE
PEOPLE TO WORK ON THIS AND ALSO
AND WE HAVE AN OPEN DOOR POLICY.
WE WANT TO HEAR FROM YOU AND
YOU'RE THE PEOPLE WE'RE WORKING
FOR AS YOU REPRESENT THE FOLKS
SUFFERING FROM NEUROLOGICAL
DISORDERS.
AND IF THERE'S THINGS THAT CAN
COME TO YOUR MIND THAT COULD
MAKE OUR JOINT ENDEAVORS MORE
SUCCESSFUL