I WANT TO WELCOME EVERYONE TO
THE SECOND DAY OF THE ALS
STRATEGIC PLANNING WORKSHOP THAT
NIH IS HOSTING.
WE'LL GET STARTED IN A COUPLE
MINUTES BUT FIRST I WANT TO
REMIND YOU WE'LL CONTINUE TO
ACCEPT ALL QUESTIONS AND
COMMENTS REGARDING THE DRAFT
PRIORITIES FOR ALS STRATEGY
PLAN.
YOU CAN SEND IN QUESTIONS AND
COMMENTS ON THE WEBINAR VIA THE
ZOOM Q&A BOX OR IF YOU'RE NOT ON
THE WEBINAR OR WANT TO USE THE
FORUM WE'LL PUT IT IN THE CHAT
AND YOU'LL ABLE TO ACCESS THIS
VE VIA THE WEBSITE.
YOU CAN E-MAIL OUR LOGISTICS
EXPERT WITH ANY QUESTIONS.
WE'RE REGARDING THE WORKSHOP
TODAY AS YESTERDAY AND THE
RECORDING WILL BE AVAILABLE
WITHIN ONE WEEK OR SO OF THE
EVENT.
NOW, I'D LIKE TO INTRODUCE OUR
STRATEGIC PLAN STEER COMMITTEE
CO-LEADS WITH DR. BROWN AND
SATTLER FOR THEM TO WELCOME US
AND ENTER INTO DAY 2.
>>GOOD MORNING.
I'D LIKE BEGIN BY WELCOMING
EVERYONE BACK TO DAY 2 OF THE
STRATEGIC PLANNING WORKSHOP.
IT'S BEEN AN INCREDIBLE DIALOG.
WE ENJOYED THE COMMENTS
YESTERDAY WHICH I HOPE CAPTURED
THE EXCITEMENT ALL OF US IN ALS
HOPE IS HERE AS WE MOVE IN THE
NEXT GENERATION OF THERAPIES
TOWARD MEANINGFUL SOLUTIONS TO
ALS.
I THINK THEY'RE CLOSE AT HAND
AND IT'S BEEN AN HONOR TO
PARTICIPATE IN THE DISCUSSION SO
FAR.
IF THERE'S ONE THING WE AGREED
ON IS THAT THESE ARE EXCITING
TIMES AND WE NEED TO FOCUS AND
BRING NEW TOOLS TO BEAR IN
UNDERSTANDING SPRAD ACHE ALS --
SPORADIC TOPICS AND TODAY WE'LL
LOOK AT AN ADDITIONAL SET OF
WORK GROUP AND DR. SATTLER WILL
REVIEW THE OUTLINE FOR THE DAY.
RITA.
>>THANK YOU.
AND WELCOME EVERYBODY.
WELCOME BACK FOR THOSE WHO
JOINED YESTERDAY AND I CAN ONLY
DITTO WHAT BOB JUST SAID.
IT WAS A WONDERFUL DAY YESTERDAY
AND REALLY INDEED THERE'S
EXCITEMENT TO FOCUS ON THE
TREATMENT AND FIND CURES FOR
THIS DEVASTATING DISEASE.
TO INTRODUCE WHERE WE'LL GO
TODAY.
YOU CAN SEE HERE IT'S JUST AGAIN
A SUMMARY OF THE WORKING GROUPS
WE HAVE PUT TOGETHER FOR THIS
EFFORT AND YESTERDAY YOU HEARD A
LOT ABOUT THE RESEARCH BEHIND
ALS AND HOW WE TRANSLATE THE
FINDINGS THAT WE HAVE IN THE
LABORATORY AND BRING THEM TO THE
CLINIC AND HOW DO WE UTILIZE
THEM IN THE CLINIC AND PERFORM
CLINICAL TRIALS FOR PATIENTS WE
CAN INTEGRATE PATIENTS NOT JUST
SYMPTOMATICALLY BUT PRESYMPTOM
ATCALLY AND WAS A TOPIC
YESTERDAY.
WHAT WE'LL FOCUS ON TODAY IS THE
PEOPLE LIVING WITH ALS.
FOR THAT WE HAVE TWO WORKING
GROUPS AND ONE TRULY FOCUSSED ON
THE QUALITY OF LIFE OF PEOPLE
WITH ALS AND THIS IS AN
IMPORTANT TOPIC FOR US BECAUSE
THERE'S A LOT THAT CAN STILL BE
DONE IN ORDER TO FACILITATE HOW
PEOPLE WITH ALS AND CAREGIVERS
GO THROUGH DAY TO DAY LIFE AND
THERE'S A LOT TO BE DONE AND
WE'LL DISCUSS IT IN DETAIL AND
WE HAVE AN INCREDIBLE WORKING
GROUP TO ADDRESS THE TOPIC.
WHAT WE WANT TO ADDRESS IS HOW
CAN WE IMPROVE AND OPTIMIZE
SYMPTOM MANAGEMENT
PHARMACOLOGICALLY AND
NON-PHARMACOLOGICALLY FOR PEOPLE
WITH ALS AND HOW CAN WE
FUNCTIONALIZE PEOPLE LIVING WITH
ALS AND THERE'S TECHNOLOGIES
ALLOWING PEOPLE LIVING WITH ALS
TO CONTINUE COMMUNICATION WITH
THE OUTSIDE WORLD EVEN IF THEY
LOSE THEIR ABILITY TO SPEAK.
THIS IS GOING TO BE A REALLY
IMPORTANT TOPIC WE NEED TO FOCUS
ON IN THE FUTURE AND FINALLY, IT
IS IMPORTANT FOR US WE CAN
IDENTIFY MORE AND NEW RESOURCES
AND AVAILABILITY OF THE
RESOURCES FOR EVERYBODY FOR
EVERY PERSON THAT LIVES WITH ALS
AND THEY'RE CAREGIVERS TO MAKE
SURE THAT ACCESS TO THESE
RESOURCES ARE THERE AND
POTENTIALLY IDENTIFY NEW
RESOURCES WE CAN PROVIDE FOR
THIS PATIENT POPULATION.
THEN IN THE LAST WORKING GROUP,
OF IF I CAN HAVE THE LAST SLIDE
AND I FORGOT TO MENTION THE
CO-CHAIRS BUT THEY'LL INTRODUCE
THEMSELVES SHORTLY AND THE
CO-CHAIRS FOR THE LAST WORK
GROUP, DR. LISA GOLD AND DAN
DOCTOROFF AND A TOPIC IS HOW DO
WE BRING EVERYBODY TOGETHER AT
THE TABLE SO THAT WE CAN MOVE
FASTER AND MORE EFFICIENTLY TO
OUR COMMON GOAL AND THIS IS
REALLY TO PROVIDE OPPORTUNITIES
FOR COLLABORATION AND FOR
PARTNERSHIPS.
AND IT WAS DISCUSSED YESTERDAY A
LOT WE NEED MORE MULTI-MODAL
PLATFORMS TO SHARE DATA AND
SHARE RESEARCH TOOLS AND
PROTOCOLS AND BIO SAMPLES AND
THAT NEEDS TO BE AVAILABLE FOR
EVERYBODY, FOR ACADEMICS, FOR
CLINICIANS, FOR INDUSTRY.
WE NEED TO FOSTER THIS CLOSE
RELATIONSHIP ESPECIALLY BETWEEN
INDUSTRY AND ACADEMIA.
THERE'S STRENGTH IN BOTH SITES
AND A LOT OF STRENGTH IN THE
INTELLECTUAL DISCOVERY WE HAVE
IN ACADEMIA AND STRENGTH IN HOW
INDUSTRY CAN GRAB THIS
INTELLECTUAL PROPERTY AND MOVE
IT FORWARD TOWARDS A MEANINGFUL
DEVELOPMENT OF THERAPEUTICS THAT
WE CAN THEN PROVIDE TO PATIENT
WITH ALS.
WE'RE EXCITED TO DISCUSS BOTH
TOPICS AS PRIORITIES IN TODAY'S
SESSION.
AND JUST LIKE YESTERDAY WE WANT
TO START OFF OUR DAY WITH WHAT
WE CALL OUR ALS LIVED EXPERIENCE
PANEL.
LIKE YESTERDAY IT WILL BE
CO-CHAIRED BY LAYNE OLIFF AND
ELLYN PHILLIPS AND LISTEN TO THE
QUESTIONS RAISED.
I'LL HAND IT OVER AND I WISH
EVERYBODY A GREAT DAY AND I LOOK
FORWARD TO MEANINGFUL
DISCUSSIONS AND COMMENTS.
THANK YOU, EVERYBODY.
>>THANK YOU, RITA AND WHAT A
GREAT DAY, YESTERDAY.
IT REALLY WAS.
GOOD MORNING, EVERYONE.
HOPEFULLY MY VOICE WILL HOLD OUT
TODAY TO THEN OF THE DAY.
WHAT WE WANT TO DO THIS MORNING
IS GIVE A COUPLE MINUTES
HIGHLIGHT AND ELLYN WILL TAKE A
QUESTION AND I'LL TAKE A
QUESTION AND WE'LL END WITH A
THANK YOU.
KEY HIGHLIGHTS FROM THE PANELS
SPECIFICALLY YESTERDAY, I WOULD
USE THE WORDS CAUTIOUSLY
OPTIMISTIC.
THE PANELISTS WERE VERY EXCITED
ABOUT THE IDEA OF AN ALS DRAFT
PLAN BUT WERE CAUTIOUSLY
OPTIMISTIC AND ON THE
IMPLEMENTATION AND THAT'S THE
KEY FINDINGS I SEE FROM THE
PANEL YESTERDAY.
ELLYN, DO YOU HAVE INSIGHT?
>>FOR SURE.
AND LAYNE THANK YOU FOR ALL YOUR
SUPPORT AND HELP.
I FEEL LIKE A NEOPHYTE THOUGH
I'M HOLDER THAN YOU AND I
APPRECIATE EVERYTHING YOU HAVE
DONE AND ALL OF THE PANELISTS
AND NINDS AS WELL.
SOME TAKEAWAYS FROM YESTERDAY,
THE PANELISTS WANT TO STAY
INVOLVED.
AND YOU CAN SEE THAT PEOPLE ARE
SO EXCITED ABOUT THE
POSSIBILITIES THAT COULD BE
BROUGHT FORTH FROM THE STRATEGIC
PLAN.
AND THE SECOND TAKEAWAY IS THAT
EVERYONE WANTS TO MAKE SURE THAT
THERE'S FUNDING AND MAKING SURE
THAT IT DOES GET FUNDED.
I BELIEVE WALTER ADDRESSED THAT
YESTERDAY.
LAYNE.
>>DO YOU WANT TO INTRODUCE THE
PANELISTS.
>>WE'RE THE FIRST TWO ON THE
PHOTOS.
THERE'S GWEN AND ED AND ANDREA
AND ALISON AND NADIA AND COOPER
AND STEVE.
I'LL ASK THE FIRST QUESTION -- I
WANT TO MENTION ABOUT ALLISON
FIRST.
>>WHY DON'T YOU DO THAT.
>>UNFORTUNATELY, ALLISON WON'T
BE ON TODAY.
SHE HAD A MEDICAL EMERGENCY WITH
HER SISTER AND THAT'S ALL WE
KNOW AS A PRIORITY.
SHE WILL NOT BE ON TODAY.
WE HAVE ANSWERS FOR HER
QUESTIONS AND WE'LL BE PREPARED
WITH THOSE.
>>THE FIRST QUESTION IS WHAT
WAS YOUR ROLE IN THE WORKING
GROUP?
COOPER.
>>I US UP STUDYING FOR AN EXAM.
THE WORKING GROUP FUNCTIONED
LIKE LAB MEETINGS I HAD.
I THINK WE ALL BROUGHT A LOT OF
TO THE TABLE.
I DON'T HAVE THE HUBRIS TO THINK
I BROUGHT ANYTHING SUPER
SPECIAL.
I THINK MY FOCUS WAS ON
PRE-SYMPTOMATIC TESTING AND WE
ALL PUT ASIDE OUR THEORIES AND
WHAT WE BELIEVE IS THE MOST
IMPORTANT TARGET BUT I FEEL THE
PRIORITIES WE PUT FORWARD ARE
BROAD AND GENERATIVE.
I WAS GRATEFUL FOR THAT AND
THOUGHT WE ALL BROUGHT A LOT TO
THE TABLE.
>>THANK YOU, COOPER.
NADIA.
>>I THINK I BROUGHT THE
COMMUNITY PERSPECTIVE TO MY
GROUP.
IT WAS A BRILLIANT GROUP OF
PEOPLE BUT HAVING THE EXPERIENCE
OF HAVING GONE THROUGH ALS OUR
CONCERNS ARE A LITTLE BIT
DIFFERENT.
I THINK THAT IS WHAT I BROUGHT
TO THE GROUP WAS SHARING THE
PERSPECTIVE OF SOMEBODY WHO'S
LIVED THE DISEASE AND THE
OBSTACLES.
I'M STILL CARRYING AROUND MY
HUSBAND'S GENETIC TESTING
RESULTS AND NO ONE WILL TAKE THE
DATA FROM ME.
PERSPECTIVES LIKE THAT THAT I
THINK ARE IMPORTANT AND MISSED.
THAT'S WHAT I BROUGHT TO MY
GROUP.
GWEN.
>>I'M GWEN PETERSEN.
I'M LIVING WITH ALS DIAGNOSED IN
2018 AND YOU HEARD ME YESTERDAY.
AND MY GROUP WAS OPTIMIZING ALS
CLINICAL RESEARCH AND WE
PRESENTED YESTERDAY AFTERNOON
WAS LEVERAGED MY KILLS AS A
FORMER PROFESSIONAL, FULL-TIME
RECRUITER AND THE BASICS I
OFFERED AND MANY OF YOU KNOW ME.
YOU KNOW I'M ALWAYS WILLING TO
JUMP IN AND OFFER ORGANIZATIONAL
SUPPORT, SCHEDULING,
CALENDARING.
IT'S JUST PART OF PROFESSIONALLY
I HAD TO BE A PROFESSIONAL WHEN
IT CAME TO THAT.
I TOOK CARE OF PRE MEETING
ACTION ITEMS AND DELIVERABLES.
AN ASSUMED THE RESPONSIBILITIES
FOR TWO REASONS.
THE COORDINATOR WAS ASSUMING
MOST THE RESPONSIBILITY AND
SHE'S BUSY.
SHE HAS A FULL CALENDAR.
I WANTED TO TAKE THAT OFF OF
HER.
AND SECOND REASON WHY I DID IT
WAS AGAIN MANY OF THE MEMBERS ON
THESE PANELS ARE ACTIVELY SEEING
PATIENTS AND I'M BIASED.
I WANT PATIENTS TO COME FIRST
AND NOT LET MINUTIA OF
SCHEDULING, CALENDARING, GET IN
THE WAY.
THAT'S THE TYPE OF SUPPORT I
BROUGHT.
>>THANK YOU, GWEN.
STEVE.
>>GOOD MORNING, EVERYBODY.
MY NAME IS STEVE AND HAPPY TO BE
HERE AND DIAGNOSED WITH ALS IN
2017 AND REACHED MY FIVE-YEAR
MARK IN LIVING WITH THIS
DISEASE.
I AM PART OF THE QUALITY OF LIFE
WORKING GROUP AND AS I SAID
YESTERDAY PATIENT LIVED
EXPERIENCE IS CRITICAL AND I
APPLAUD THE NIH FOR INCLUDING
ALL OF US PALS AND GALS IN
PROVIDING OUR INPUT.
LIKE GWEN SAID OUR LIVED
EXPERIENCES ARE TWOFOLD.
THEY'RE PRIOR TO ALS AND PENN IR
LIVED EXPERIENCE AFTER ALS.
FOR ME I SPENT 34 YEARS IN THE
HIGH TECH WORLD AND BRING A
SECURITY SCIENCE ASPECT TO THIS
CONVERSATION AS WELL AS MY
PERSONAL EXPERIENCE.
WHEN I STARTED THIS ENDEAVOR I
DUG DEEP IN THE 300 RESPONSES TO
THE RFI WE ALL RECEIVED.
I WANTED TO GO THROUGH EACH ONE
OF THOSE AND PARS OUT WA WHAT
WAS IMPORTANT IN TERMS OF
QUALITY OF LIFE AND I WAS ABLE
TO BRING THIS TO A TALENT THE
GROUP OF PEOPLE AND THINK OF
WAYS TO IMPROVING QUALITY OF
LIFE AND A LOOK FORWARD TO THAT
PRESENTATION AFTER WE HAVE OUR
PANEL DISCUSSION RIGHT NOW.
THANKS.
>>THANK YOU.
ED.
>>I WAS DIAGNOSED IN 2015.
I'D SAY MY ROLE IN THE SUBGROUP
WAS TWOFOLD.
FIRST WAS TO LISTEN AND LEARN.
I KEEP A LIST OF THE GIFTS OF
ALS ON MY PHONE AND ONE IS THE
AMAZING NETWORK OF PEOPLE I'VE
MET ON THIS JOURNEY.
MY SECOND ROLE WAS TO CHAMPION
THE CONCEPT OF THE NEED FOR BIG
DATA ANDIO FLUID -- BIO FLUID
IN LABORATORIES.
UNTIL YOU UNDERSTAND ROOT CAUSE
ANY SOLUTION IS JUST A BAND-AID.
I REMAIN CONVINCED UNTIL WE
UNDERSTAND THE SUBGROUPS OF THE
DISEASE, ANY DRUG TRIAL HAS THE
RISK OF BEING A SHOT IN THE
DARK.
SO MY JOB WAS TO CHAMPION THAT
CONCEPT AS PART OF THE SUBTEAM
AND AS I SAID EARLIER TO LEARN
FROM A BUNCH OF GREAT
COLLEAGUES.
>>THANK YOU.
>>IN THE PARTNERSHIP AND
COLLABORATIONS WORKING GROUP AND
IF YOU KNOW ME YOU KNOW IT'S A
TRUE PASSION OF MINE.
I'M NOT SHY.
I'LL TALK TO ANYBODY WHO I THINK
CAN PULL TOGETHER THE
INFORMATION WE NEED AND MAY HAVE
THE EXPERTISE TO HELP US MOVE
OUR OBJECTIVES SO FORWARD.
MY ROLE IN THE WORKING GROUP WAS
AN A CAREGIVER AND ALS
PROFESSIONAL AND WORKED TWO
PRIORITIES.
ONE WAS TO FOCUS ON INCREASING
THE DIVERSITY OF RESEARCH
DATABASES USING A HEALTH EQUITY
LENS AND SECONDLY WAS TO
ESTABLISH A COMPREHENSIVE
RESEARCH PROJECT LANDSCAPE.
ONE OF THOSE PRIORITIES MOVING
FORWARD ON THE HEALTH EQUITY
LENS FRONT WAS TO HELP MEME WITH
ALS HAVE BETTER ACCESS TO
RESOURCES AND CLINICAL TRIALS
AND THEN WE CAN IMPROVE THE
DIVERSITY OF WHO'S IN THE
RESEARCH DATABASES.
THAT MERGED WITH THE QUALITY OF
LIFE GROUP WHICH IS TERRIFIC.
AS LONG AS IT'S IN THE PLAN IT'S
GREAT.
I DIDN'T MATTER WHO'S TAKING
CREDIT FOR IT, WE'RE ALL IN THIS
TOGETHER.
THE OTHER PIECE THAT WAS
IMPORTANT TO ME IN THE WORK
GROUP AS A THIRD PRIORITY WAS TO
DEVELOP A COMPREHENSIVE RESEARCH
LANDSCAPE BECAUSE IF WE CAN'T
IDENTIFY ALL THE AREAS IN WHICH
RESEARCH IS TAKING PLACE AND WHO
IS DOING THAT RESEARCH HOW ARE
WE GOING TO COLLABORATE?
I FELT THOSE WERE THE TWO PIECES
I WAS ABLE TO BRING AND WAS
INCREDIBLY PLEASED WE WERE IN A
HIGH QUALITY, DEDICATED GROUP OF
PEOPLE.
>>THANK YOU, ANDREA.
LAYNE WOULD YOU LIKE TO READ
ALLISON'S SUBMISSION?
>>I WILL.
I WAS A PART OF A REALLY GREAT
WORKING GROUP WHICH I BELIEVE
WAS MADE OF INDIVIDUAL
CONTRIBUTORS BROUGHT GEM BECAUSE
OF THEIR EDUCATION, EXPERTISE
AND EXPERIENCE AND PERSPECTIVE.
BASED ON THAT I TRIED TO BRING
THE PERSPECTIVE OF MANY COMBINED
VIEWS.
OF COURSE MY OWN LICHED
EXPERIENCES HAVING TRAVELED THE
ALS PATHWAY WITH MY HUSBAND BUT
BEING VERY ACTIVE IN THE
COMMUNITY AT LARGE HAS TAUGHT ME
THAT WHILE WE ALL MAY SHARE
THINGS SUCH AS PAIN, GRIEF AND
LOSS, OUR EXPERIENCES AND
OPTIONS WHEN MANEUVERING OUR
LIFE WHEN IMPACTED BY ALS CAN BE
VASTLY DIFFERENT.
IN TALKING WITH FAMILIES
IMPACTED BY ALS THROUGH MY WORK
I HAVE BEEN EXPERIENCING THE ALS
JOURNEY IN MANY DIFFERENT FORMS
WITH VARYING RESOURCES,
DIFFERENT LEVELS OF KNOWLEDGE
ABOUT BOTH RESEARCH OPTIONS.
I'VE SEEN MANY DIFFERENT FAMILY
DYNAMICS AT PLAY, DIFFERENT
SUPPORT SYSTEMS AND DIFFERENT
FINANCIAL CAPABILITIES ALL
COMBINED WITH A HORRIFIC MENU OF
SYMPTOMS THAT CAN BE PRESENT IN
MANY COMBINATIONS AT ANY TIME.
AND THOUGHT MY POSITION WOULD BE
A SPONGE AND SEEK AND LEARN AND
UNDERSTAND THE NEEDS OF ALL OF
THOSE IMPACTED BY ALS AND TO
FUNNEL THAT KNOWLEDGE INTO INPUT
AND PERSPECTIVE THAT CAN HELP
MAKE DECISION ARE GOOD DECISIONS
FOR THE COMMUNITY AS A WHOLE,
FOR PATIENTS, FOR CAREGIVERS AND
FAMILY MEMBERS AND ALL THE
PEOPLE WITHIN THE ORGANIZATION
THAT SUPPORT THEM.
THANK YOU.
>>THANK YOU, LAYNE.
>>OH, DID YOU WANT TO ADD
ANYTHING BEFORE I GET TO THE
LAST QUESTION?
>>I WOULD LIKE TO SAY THIS HAS
BEEN AN INCREDIBLE EXPERIENCE.
I HAVE SERVED ON NINDS COUNCIL.
THAT OPENED MY EYES TO A LOT OF
THINGS AND THAT WAS SEVERAL
DECADES AGO.
AS MEMBERS OF THE STEERING
COMMITTEE WE CAN LISTEN AND SIT
AND PARTICIPATE ON ANY OF THE
WORKING GROUPS.
I WAS VERY CONCERNED ABOUT THE
READABILITY OF THE DOCUMENT AND
GIVE KUDOS TO PEOPLE WHO
EVERYONE WHO HAD A HAND IN
WRITING THIS BECAUSE IT WAS EASY
TO READ ESPECIALLY THE
NON-TECHNICAL SUMMARY.
I THINK PEOPLE WILL APPRECIATE
THAT VERY MUCH.
>>GREAT COMMENT.
THERE IS A FOCUS IN THE
NON-SCIENTIFIC ALS COMMUNITY IN
THIS DOCUMENT.
IT'S SO IMPORTANT.
AND LET ME ASK WHAT ONE OR TWO
KEY TAKEAWAYS AND WOULD YOU HAVE
FUND THIS PLAN THE ALS STRATEGIC
PLAN.
COOPER, I'LL MAKE YOU START.
>>THANKS, LAYNE.
I'M VERY EXCITED ABOUT THIS PLAN
AND SOMETHING WONDERFUL I SAW
YESTERDAY I THINK REFLECTS
FOCUSES IN OTHER AREAS.
I FELT A LOT OF SYNERGY AND THAT
MAKES ME THINK WE'RE ON THE
RIGHT TRACK.
JUST GOING DOWN THE LIST I THINK
A BROAD THEME IS THAT WE WERE
FOCUSSED ON MAKING SURE PEOPLE
DIDN'T JUST LOOK WHERE THE LIGHT
IS BEST.
PUSHING THROUGH SOME SCIENTIFIC
INERTIA THAT HAS EXISTED.
FIRST AND FOREMOST WE NEED TO
FIGURE WHAT'S GOING ON WITH
PREP.
IT'S A HETEROGENEOUS DISEASE.
WE NEED GOOD MODELS AND NEED A
WAY TO TARGET THIS AND
UNDERSTAND IT AND INCENTIVIZE
PEOPLE TO LOOK AT THE QUESTIONS.
I PRAY THE FUNDING WILL COME.
I THINK WE SET OUT TOOLS AND
GROUND WORK TO ASK SCARY
QUESTIONS.
IT'S HARD.
SPEAKING AS SOMEONE WHO THINK
ABOUT THIS ALL DAY EVERY DAY AND
WORKS IN THE LAB IT'S HARD TO
DESIGN QUESTIONS AROUND THIS AND
I THINK WE'VE INCENTIVIZED
PEOPLE TO DO SO AND HOPE IT WILL
BE EFFECTIVE.
TWO, IS UNDERSTANDING CLINICAL
HETEROGENEITY.
HOW ARE YOU THINKING ABOUT
CONVERSION AND PRE-SYMPTOMATIC
CONVERSION AND RESISTANCE.
DIVING IN AND EXPAND THE
CLINICAL SPECTRUM TO INCLUDE
PEOPLE WHO DON'T NECESSARILY
HAVE MOTOR WEAKNESS AS OF YET.
THERE'S A GROWING CONSENSUS THIS
IS THE NECESSARY THING TO DO
AFFECT THE DISEASE MEANINGFULLY
AND THAT UNDERSTANDING COMES
FROM PATIENT ADVOCACY IN NO
SMALL PART.
EVERYONE LISTENING WHO'S PLAYED
A PART ON THIS GIVE YOURSELF A
PAT ON THE BACK OR ROUND OF
APPLAUSE BECAUSE NEVER AGAIN
WILL WE MOVE IN THE INERTIA OF
NOT INCLUDING THESE PEOPLE IN
THE CONVERSATION AND IN THE BIO
BANK AND REPOSITORIES AND
INCLUDING NEM IN THE MODELS OF
DISEASE.
I FEEL STRONGLY AND SOMETHING I
WAS ADVOCATING FOR AND WAS MY
AGENDA IN THE GROUP AND I THINK
THAN MANIFESTED AND SHOULD BE
PART OF THAT.
THREE IS COMMUNITY BUILDING AND
LEVERAGE.
WE TALKED ABOUT DATA
INFRASTRUCTURES.
WE HAD A SEPARATE MEETING TO
DISCUSS THE BEST WAY TO
FACILITATE THIS AND WE TALKED
ABOUT WHAT TRAININGS MAY BE
NECESSARY TO BRING PEOPLE IN THE
FIELD AND EXCITED AND EVERY DAY
THERE'S NEW TECHNIQUES COMING
OUT AND SOMEONE WILL LOOK AT THE
PATHWAYS WITH A NEW EYE AND FIND
SOMETHING NEW.
IN THAT WAY I FEEL EXCITED.
IT'S A FOCUS ON INCLUSION ACROSS
PATIENTS AND RESEARCHERS AND
WHEN WE BROADEN OUR UMBRELLA AND
PERSPECTIVE I CAN'T GO WRONG.
I FEEL GOOD ABOUT THE DECISIONS
WE MADE AND ULTIMATELY IT WILL
SERVE THE COMMUNITY IF WE GET
THE SUPPORT WE NEED.
>>THANK YOU, COOPER.
NADIA, ONE OR TWO KEY TAKEAWAY.
.
>>I HAVE TWO.
YESTERDAY THERE WAS DISCUSSION
FROM THE GEN ET AND FAMILIAL ALS
COMMUNITY AND I THINK IT GETS
LOST ON THOSE LIVING WITH ALS
AND CAREGIVERS IT'S ALL
INTERRELATED.
THE THINGS WE LEARN FROM PEOPLE
PRE-SYMPTOMATIC AFFECT ALL KINDS
OF ALS AND IF WE FIND BIOMARKERS
IT WILL WILLING HELP THE
FAMILIAL COMMUNITY AND ALL THE
TERMINOLOGY IS PROBLEMATIC AND
HAS ISSUES, SPORADIC, FAMILIAL
AND GENETIC HAS SOME WEAKNESSES
BUT ALL MOVES US FORWARD AND
DON'T WANT THAT PIECE TO GET
LOST BECAUSE THERE'S SO MUCH
GOOD NOISE BY THE FAMILIAL AND
GENETIC ALS COMMUNITY YESTERDAY
AND ADVANCES THINGS FOR ALL OF
US AND THAT SAID WE NEED TO PARS
OUT WHAT IS GOING ON WITH
SPORADIC ALS AS WELL BUT IT ALL
MOVE US FORWARD.
MY SECOND POINT IS AS LONG AS
I'VE BEEN INVOLVED IN ALS WORLD
FOR THE LAST THREE YEARS THERE'S
BEEN A STRONG CALL TO NOT
RECREATE THE WHEEL, TO BREAKDOWN
SILOS FOR COLLABORATION AND ON
THAT NOTE, THERE WERE MANY
INTERESTING IDEAS BROUGHT UP
YESTERDAY.
ONE BEING A NATURAL HISTORY
STUDY AND THERE ARE ONGOING
NATURAL STUDIES FOR ALS AND IT'S
COMMUN
IMPORTANT TO LEVERAGE THAT IN
THE COMMUNITY MOVING FORWARD IN
THE STRATEGIC PLAN.
IT WILL HELP THINGS MOVE FASTER
FOR ALL OF US AND WE SHOULDN'T
BE RECREATING WHEELS AT THIS
POINT.
THOSE ARE MY TWO TAKEAWAYS.
>>THANK YOU, NADIA.
>>I'M GLAD YOU CALLED ON ME SO
I CAN BUILD ON NADIA'S SENTIMENT
ON LEVERAGING CURRENT ASSETS.
ONE THING MY GROUP, WHICH WAS
THE CLINICAL ALS RESEARCH, ONE
THING AMONG MANY THAT WE TALKED
ABOUT WAS LOOKING AT THE CURRENT
LANDSCAPE AND THE BEST OF THE
BEST AND WHAT EXISTS IN TERMS OF
NATURAL HISTORY STUDIES.
THE BEST OF THE BEST AND WHERE
THE GAPS ARE AND HOW TO GO ABOUT
FILLING THOSE GAPS.
SO I THINK WE DID A REALLY GOOD
KIND OF COMPREHENSIVE LOOK AT
THE LANDSCAPE AND BUILT FROM
THERE.
ALSO, I'M NOT IN THE BUSINESS OF
REPEATING NADIA AND COOPER, YOU
GUYS SAID ALL GREAT THINGS.
ONE THING IS I WOULD WANT THE
LARGER COMMUNITY TO KNOW THAT
PEOPLE LIVING WITH ALS, LOVED
ONES ARE FAMILIAL ALS FRIENDS,
ANYONE SERVING IN THE WORKING
GROUPS HAD A TON TO DO WITH THIS
STRATEGIC PLAN.
OUR FINGERPRINTS ARE ALL OVER
TH
THIS.
WE STEPPED UP AS LEADERS IN THE
COMMUNITY AND OUR CO-LEADS
WELCOMED US TO STEP UP WITH OUR
THOUGHTS AND IDEAS SO I JUST
WANT THE COMMUNITY TO KNOW THAT
THIS DOCUMENT VERY VERY MUCH
BUILT BY THE PEOPLE IN SQUARES
THAT YOU'RE SEEING RIGHT HERE.
SO THANK YOU.
>>GREAT COMMENTS, STEVE, YOU'RE
UP.
THE TECH GUY --
>>YOU'D THINK I'D FIGURE THIS
OUT BY NOW.
I AGREE WITH EVERYTHING SAID SO
IT'S HARD GOING AT THE END
BECAUSE YOU FIND THINGS YOU HAVE
TO ADD THAT HAVEN'T BEEN ALREADY
SAID.
MY LIVED EXPERIENCE IS I'VE BEEN
INVOLVED IN MANY STRATEGIC
PLANNING EFFORTS AND I CAN TELL
YOU THOSE WELL CRAFTED AND WELL
DONE GET FUNDED AND I THINK
WE'RE ON A PATH TO TO BATCH WITH
THIS PLAN.
I ALSO WOULD REMIND EVERYBODY
WHAT THIS PLAN DOES AS IT EXISTS
RIGHT NOW EVEN TO HELP THOSE
NEWLY DIAGNOSED.
I WOULD HAVE LOVED TO HAVE READ
THIS ON DAY TWO AFTER I RECEIVED
THE DIAGNOSIS OF ALS.
IT WOULD HAVE GIVEN ME HOPE AND
INSPIRED ME IN WAYS I DIDN'T
FEEL AT THE TIME THERE'S A LARGE
GROUP OF DEDICATED AND
PASSIONATE PEOPLE WORKING EVERY
DAY TO MAKE THIS A BETTER
EXPERIENCE AND ULTIMATELY GET TO
A CURE.
YESTERDAY I REFLECTED UPON SOME
OF THE COMMENTS THAT SAID
PERHAPS THIS WAS A LITTLE TOO
AMBITIOUS.
A LITTLE TOO LARGE IN SCOPE AND
MY THOUGHT ON THAT IS THAT THE
RESPONSE SHOULD BE THIS IS STILL
THE WORK IN PROGRESS.
THE WORD DRAFT MEANS SOMETHING.
IT MEANS THAT WE NEED FURTHER
FEEDBACK AND INPUT FROM PALS AND
GALS TO STRENGTHEN THIS AND HOPE
THOSE IN THE POSITION TO DO THAT
TAKE THAT ADVICE MOVING FORWARD.
THANKS.
>>THANK YOU, STEVE.
NEXT, ED.
>>STEVE, TO YOUR POINT,
EVERYONE HIT ON THE KEY COMMENTS
BUT CONSISTENCY FROM THE ALS
COMMUNITY IS KEY HERE.
FOR ME, IF I LOOK AT THE PATIENT
AND CAREGIVERS WE NEED TO
RECOGNIZE ADVOCACY ON OUR BEHALF
DOES MATTER AND WE NEED TO STAY
ENGAGED.
THE ACT FOR ALS AND APPROVAL OF
THE DRUG OR THE FORMATION OF THE
STRATEGY, PATIENTS AND
CAREGIVERS PARTICIPATION HAS
MADE A DIFFERENCE.
SO WE NEED TO STAY ENGAGED.
THE SECOND TAKEAWAY IS THE NIH
HAS DONE TWO THINGS.
THEY RAISED HOPE BY THE DEVELOP
THE OF THE PLAN AND ALSO RAISED
OUR EXPECTATIONS IN TERMS OF
WHAT NIH WILL DO WITH IT.
I THINK IN TERMS OF
ACCOUNTABILITY, WE AS A PATIENT
COMMUNITY HAVE A RESPONSIBILITY
TO JUST KEEP PUSHING.
THOSE WOULD BE MY TWO KEY
TAKEAWAYS.
>>GREAT COMMENTS.
ANDREA.
>>I HAVE TWO TAKEAWAYS.
I DON'T THINK EITHER OF THESE
HAVE BEEN SAID.
ONE HAS TO DO WITH NEXT STEPS
AND ONE HAS TO DO WITH TIMING.
THIS WORK CANNOT END WHEN WE
STEP AWAY FROM OUR COMPUTER
TODAY AND WE HAVE 52 MEMBERS AND
THE NINDS AND NIH MEMBERS AND
CANNOT AND WILL NOT AND LET THE
THOUSANDS OF HOURS OF WORK WE
PUT IN THIS PLAN GO TO WASTE.
SO STEVE, YOU'RE RIGHT, THIS IS
A HUGE BOLD PLAN.
NO DOUBT.
IT WILL NEED TO BE IMPLEMENTED
WITH INTENTION AND FORETHOUGHT.
MY SECOND POINT IS THIS -- I'M
GOING KEEP IT TOGETHER A MOMENT.
I WANT TO REALIZE THE STARK
REALITY OF WHY WE DO THIS.
ALL OF US ON THIS PANEL ARE
EITHER LIVING WITH ALS OR LOVED
SOMEONE WITH ALS AND SINCE THE
PLAN WAS ANNOUNCED IN JANUARY OF
THIS YEAR WE'VE LOST OVER 5,000
PEOPLE TO ALS IN THE UNITED
STATES.
WE ABSOLUTELY HAVE TO BE ON THE
ALS CLOCK FOR THOSE WE'VE LOST
TO ALS AND THE CARRIERS AND
THOSE LIVING WITH ALS.
>>GOOD THINGS ARE HAPPENING AND
THEY'LL CONTINUE TO HAP JPEN AN
THERE ARE AREAS TO IMPROVE UPON
TO INCREASE THE SPEED OF SOME
PROCESS, PROJECTS AND PROCESSES.
BUT IT'S IMPORTANT TO KNOW THIS
ISN'T ALWAYS DUE TO LACK OF
CARING OR TRYING.
SO MANY HAVE GIVEN SO MUCH OF
THEIR TIME TO IDENTIFY THE MOST
IMPORTANT CHANGES WE CAN MAKE AS
A COMMUNITY.
MY TAKEAWAY FOR YOU TO PLEASE
KNOW HOW HARD PEOPLE ARE WORKING
TO HELP US AND MY TAKEAWAY OF
THOSE DIRECTLY IMPACTED BY ALS,
I'M NOT SURE I CAN DO JUSTICE IN
EXPLAINING TO YOU HOW IT CAN
FEEL LIKE AN ETERNITY ON THE
OUTSIDE OF YOUR PERFECTLY LAID
PLAN AND HOPE NIH WILL CONTINUE
TO BE FORTHRIGHT AND SPEND TIME
IN ACTION AND MAKING THOSE
THINGS HAPPEN.
HOPEFULLY DURING THE SESSIONS
HAVE YOU GROWN TO KNOW US AS
REAL PEOPLE AND NEED TO BE
FLEXIBLE AND TAKE RISKS ON OUR
BEHALF.
THIS IS NECESSARY AND
APPRECIATED WHEN I WAS SITTING
IN THE MEETINGS I CAN FEEL IT
FROM THE NIH TO THE DIFFERENT
WORK GROUPS AND CLINICIANS AND
RESEARCHERS AND SCIENTISTS AND
DIFFERENT ORGANIZATIONS INVOLVED
AND THE PEOPLE WITH LIVED
EXPERIENCE AT THE TABLE MAKING
THE IMPORTANT DECISIONS.
WE ARE ALL COMMITTED TO POSITIVE
CHANGE AND TO DOING IT TOGETHER.
IT ISN'T JUST WORDS AND ISN'T AN
AFTER THOUGHT.
IT'S WHERE OUR COMMUNITY IS
HEADED AND WE'RE DEFINITELY
PICKING UP SPEED.
>>MOST OF WHAT I SAID HAS BEEN
SAID.
I GET THE SENSE VERY MUCH THAT
NINDS IS FULLY ENGAGED AND THAT
THIS GROUP AND EVERYONE
LISTENING WILL NOT LET THIS SIT
ON A SHELF.
>>I WANT TO THANK EVERYBODY WHO
MADE THIS POSSIBLE AND CONTINUE
TO WORK HARD TO BRING THIS PLAN
TO THE MASSES AND TO SEE IT TO
FRUITION.
>>THANK YOU.
>>MY KEY TAKEAWAY MANY OF YOU
TOUCHED ON IT BUT I MENTIONED IT
YESTERDAY I WANTED TO REITERATE
11 OUT OF 52 MEMBERS OF THIS
DRAFT PLAN OVER 20% ARE PEOPLE
WHO ARE ALS EXPERIENCED EXPERTS
AND ARE GENE CARRIERS AND THAT
IS UNPRECEDENTED IN THE FEDERAL
PLAN PROCESS.
WE ARE A ROLE MODEL THAT I HOPE
OTHER FEDERAL AGENCIES WILL
FOLLOW.
NOT JUST IN THE ALS BUT OTHER
NEURODEGENERATIVE DISEASES.
NINDS WHEN THEY STARTED THE
WHOLE PROCESS SAID WE'D HAVE
PATIENT INPUT.
THEY DIDN'T JUST TALK THE TALK,
THEY WALKED THE WALK.
I THINK THAT'S AN IMPORTANT
PERSPECTIVE.
SINCE WE HAVE A LITTLE BIT OF
TIME BEFORE I DO THE THANK YOU,
WE DO HAVE ONE QUESTION, ED AND
I THINK ANDREA MENTIONED IT
TOO -- I WOULD LIKE FOR YOU TO
DISCUSS HOW THIS STRATEGIC PLAN
COULD BE ACTIONABLE.
I KNOW BOTH OF YOU HAD SOME
CONCERNS YESTERDAY.
HOW CAN WE MAKE IT ACTIONABLE?
>>IN A WORLD OF BUSINESS
STRATEGY DEVELOPMENT IS
RELATIVELY EASY.
IT'S ALWAYS THE IMPLEMENTATION
THAT'S THE HARD PART-ONE OF MY
FAVORITE BOOKS IS VICTOR PRONCO
MAN SEARCH FOR MEANING AND MY
FAVORITE LINE IS CHOICE, THE
LAST OF THE HUMAN FREEDOMS.
I THINK TO MAKE THIS PLAN
ACTIONABLE, THE NIH HAS AN
INCREDIBLY DIFFICULT TASK.
HOW DO YOU TAKE TWO DAYS OF
INCREDIBLE IDEAS FROM PASSIONATE
AND DEDICATED PEOPLE THEN BOIL
IT DOWN TO THE THINGS TO MAKE A
DIFFERENCE AND A -- SOMEONE
SAID YOU CAN'T THINK OF FINDING
A CURE.
YOU HAVE TO THINK OF IT AS A
GAME OF DOMINOS.
HE SAID HISTORY HAS KNOCKED DOWN
A NUMBER OF DOMINOS THAT LED TO
THE SCIENCE REWE REVIEWED AND
GENOME SEQUENCE GOING FROM A
MILLION DOLLARS TO A THOUSAND
DOLLARS AND THE ABILITY TO STORE
DATA IN THE CLOUD AND CRISPR
TECHNOLOGY AND THE ABILITY TO
DRAW BLOOD AND MAKE A MOTOR
NEURON.
HE SAID THOSE DOMINOS ARE DOWN
AND WHAT ARE THE ONES IN FRONT
OF YOU AND FOR ME IT'S FOR THE
NIH TO DETERMINE THE TWO TO
THREE KEY DOMINOS THAT HAVE TO
FALL TO LEAD TO A CURE.
ONCE YOU DETERMINE THOSE YOU
STAFF IT AND RESOURCE AND CREATE
A GOVERNANCE STRUCTURE THAT
DRIVES PACE AND CADENCE AND HOLD
YOURSELF ACCOUNTABLE.
IT'S THE WAY I GOT THINGS DONE
IN A GLOBAL MULTI-NATIONAL AND I
THINK THOSE PRINCIPLES WOULD
APPLY IN THIS CASE AS WELL.
THOSE WOULD BE MY THOUGHTS.
>>GREAT COMMENTS, ED.
AND-DREA.
>>ED, I 100% AGREE ON THE
BUSINESS FRONT BUT I THINK I
WANT IT ADD ONE MORE PIECE TO
THAT.
IN MY DECADES OF BUSINESS
EXPERIENCE AS WELL WE SOLVED
PROBLEMS BECAUSE WE KNEW WHAT WE
WERE GOING FOR.
WE HAVE A SITUATION WITH ALS
WHERE THERE'S SO MANY UNKNOWNS.
WE'RE LOOKING TO SOLVE SOMETHING
THAT'S NEVER BEEN SOLVED SO I
THINK WE NEED TO ADD TWO THINGS.
ONE IS A LEVEL OF FLEXIBLE.
WE CAN LEG OUT AN IMPLEMENTATION
PLAN AND LOOK AT THE ORDER IN
WHICH WE WANT TO OPERATION IZE
VARIOUS ASPECTS AND SOMETHING
UNEXPECTED WILL COME UP.
THERE WILL BE A NEW FINDING OR
TECHNOLOGY AND SOMETHING TO
CREATE DISRUPTION IN OUR PLAN
AND WE NEED TO EMBRACE IT AND
LOOK AT THAT AND BE FLEXIBLE AND
SAY THIS IS ANOTHER PATHWAY.
WE MAY HAVE NOT CONSIDERED.
I'M ENCOURAGED US TO KEEP AN
OPEN MIND BUT RESPECT TO A
DYNAMIC SITUATION.
>>THANK YOU, ANDREA.
WE WANT TO THANK YOU NIH AND
NINDS FOR DOING WHAT THEY SAID
INCLUDING US IN THE PROCESS AND
HOPE THIS WILL BE USED BY OTHER
FEDERAL AGENCIES TO EVENTUALLY
CURE ALS AND OTHER
NEURODEGENERATIVE DISEASES.
WE ALSO WANT TO THANK THE ALS
COMMUNITY AS A WHOLE.
THERE WERE OVER 300 RESPONSE TO
THE RFI.
THANK YOU, ALS COMMUNITY.
IT'S NOW OUR JOB AS A COMMUNITY
TO SUPPORT THOSE INITIATIVES
THAT WILL BE INLINE WITH THE
STRATEGIC PLAN.
IT'S BEEN SHOWN THE COMMUNITY IS
READY TO ASSIST IN ACHIEVING THE
GOALS OF THIS PLAN.
WITH THAT, I KNOW WE'RE A LITTLE
EARLY AGAIN AND WOULD LIKE TO
TURN IT OVER TO THE CO-CHAIRS OF
THE OPTIMIZING THE QUALITY OF
LIFE FOR PERSONS WITH LIVING
WITH ALS WORKING GROUP FOR OUR
NEXT SESSION.
THANK YOU.
>>THANK YOU, EVERYONE.
I'M TERRY HEIMAN-PATTERSON AND
THERE'S JEAN SWIDLER FROM THE
QUALITY OF LIFE WORKING GROUP
AND WHAT AN INSPIRATION THE
LIVED EXPERIENCE WORKING GROUP
IS TO HIGHLIGHT THE URGENCY OF
WHAT WE'RE DOING HERE IN
DEVELOPING THE STRATEGIC PLAN
AND PUSHING FORWARD FOR FUNDING.
BEFORE I INTRODUCE OUR WORKING
GROUP I WANT TO REMIND EVERYBODY
THE WORKSHOP ATTENDEES CAN ENTER
QUESTIONS IN THE ZOOM Q&A BOX
AND ENTER QUESTIONS AND COMMENTS
FOR THE WEBSITE.
SO PLEASE DO.
WE HAVE SOME QUESTIONS AT THE
END WERE ALREADY SUBMITTED.
I WANT TO TELL YOU WHAT AN HONOR
IT'S BEEN TO CONTRIBUTE TO THIS
STRATEGIC PLAN AND DEVELOPING
THE PRIORITIES THAT WILL IMPACT
THE QUALITY OF LIFE.
AND TO WORK WITH THE WORKING
GROUP THAT I WORKED WITH WAS
REALLY A PRIVILEGE.
MY COLLEAGUES, ALL OF WHOM WERE
LIKE-MINDED AND FOCUSSED ON
DOING THE BEST JOB POSSIBLE TO
BRING THE PRIORITY TO LIFE.
EVERYONE IN THE GROUP WAS
COMMITTED AND REALLY CONTRIBUTED
MAJ -- MAJORLY TO THE
PRIORITIES AND MY CAREER HAS
BEEN SPENT TAKING CARE OF PEOPLE
LIVING WITH ALS AND DOING
CLINICAL AND BASIC RESEARCH.
THAT CAREER IS NOW 40 YEARS AND
IT'S HARD TO BELIEVE I'D LIKE
ASK MY CO-CHAIR TO INTRODUCE
HERSELF AND WE'LL GO THROUGH
EVERYBODY.
>>THANK YOU.
I'M JEAN SWIDLER THE GENETIC ALS
ADVOCATE AND I HAVE THE REPEAT
EXPANSION AND MY GREAT
GRANDFATHER AND GRANDMOTHER DIED
OF ALS.
AND MY AUNT IS LIVING WITH ALS
NOW AND I HAVE 12 AUNTS, UNCLES,
SIBLINGS WHO HAVE A 50/50 SHOT
OF HAVING THE MUTATION.
I DON'T KNOW WHO WOULD LIKE TO
INTRODUCE THEMSELVES NEXT OR
WILL YOU CALL FOLKS OUT?
>>SURE.
DR. ANDREWS ARE YOU ON?
>>I'M JINSY ANDREWS AT COLUMBIA
UNIVERSITY.
I'M A CLINICAL NEUROLOGIST AND
ALS SPECIALIST AND CO-CHAIR.
>>DR. CARTER ARE YOU AVAILABLE?
>>HI, I'M CHELSEY CARTER AT IN
THE SCHOOL OF PUBLIC HEALTH AT
YALE UNIVERSITY AND RESEARCHER
IN THE ALS BASE AND VOLUNTEER
AND ADVOCATE FOR ALS SINCE I WAS
15.
THANKS FOR HAVING ME.
>>THANK YOU.
>>GOOD MORNING.
I'M MELANIE FRIED-OKEN AND I
WORK AT OREGON UNIVERSITY AND A
HAVE A 40 YEAR WORKING WITH PALS
IN THE AREA OF ASSISTIVE
COMMUNICATION AND KNOWN AS
ALTERNATIVE COMMUNICATION.
CURRENTLY, WITH A 15-YEAR LAB
LOOKING AT BRAIN COMPUTER
INTERFACES.
>>THANKS, MELANIE.
>>HI, EVERYONE, AGAIN.
I INTRODUCED MYSELF IN THE
PREVIOUS SECTION BUT MY NAME IS
STEVE AND I'VE BEEN LIVING WITH
ALS SINCE 2017.
PRIOR TO ALS I SPENT MY CAREER
IN TECH WORKING FOR A SMALL
COMPANY IN CAN CUPERTINO CALLED
APPLE.
YOU MAY HAVE HEARD OF IT.
I LIVE HERE IN BOSTON,
MASSACHUSETTS AND AM NOW RETIRED
AND DEDICATE MY WAKING HOURS TO
ALS ADVOCACY.
>>THANK YOU, STEVE.
>>I'M EMILY PLOWMAN.
I'M A PROFESSOR AT THE
UNIVERSITY OF FLORIDA IN
GAINSVILLE, GO GATORS.
AND TRAINED AS A SPEECH LANGUAGE
PATHOLOGIST BUT REALLY
PASSIONATE ABOUT DEVELOPING
PROACTIVE MANAGEMENT APPROACHES
FOR IMPROVING AND OPTIMIZING
BREATHING FUNCTIONS, AIRWAY
CLEARANCE AND SWALLOWING
FUNCTION AND I ACTUALLY DO HAVE
A NATURAL HISTORY STUDY FUNDED
BY THE NINDS LOOKING AT
PROGRESSION OF DYSFUNCTION OVER
THE COURSE OF THE DISEASE AND
IT'S A TRUE HONOR TO BE HERE.
THANK YOU VERY MUCH.
>>DR. SIMMONS WAS UNABLE TO BE
HERE AND MUCH OF HIS RESEARCH
HAS BEEN IN QUALITY OF LIFE
METRICS.
HE WAS INVALUABLE AND
CONTRIBUTED TO ANSWERING SOME OF
THE QUESTIONS YOU'LL HEAR LATER.
AND DR. HETTINGER WHO KEPT US
TOGETHER.
>>
>>I WON'T BE INTRODUCING
MYSELF.
>>SHE'S IS IN THE BACKGROUND
BUT SHE KEPT US TOGETHER.
WE APPRECIATE THE NIH SUPPORT
AND WORKING GROUP.
I'D LIKE TO ASK JEAN SWIDLER TO
KICK IT OFF WITH A PERSON STORY
OF HOW TECHNOLOGY HAS IMPACTED
HER FAMILY'S QUALITY OF LIFE.
JEAN, GO AHEAD AND START.
>>THANK YOU.
I INTRODUCED MYSELF AND YOU
HEARD MY FAMILY MEMBERS HAVE
BEEN IMPACTED BY ALS AND IT'S
QUITE LIKELY TO BE IN MY FUTURE
AND I'M SURE IN A NUMBER OF MY
ATLAS RELATIVES AS WELL.
AS AN ASIDE I WANTED TO MAKE A
NOTE I THANK THE NIH FOR BEING
INTENTIONAL ABOUT INCLUDING US
JUST LIKE EVERY OTHER ALS
ORGANIZATION WHEN THE PLAN WAS
FIRST ANNOUNCED, OUR COMMUNITY
WAS NOT ALL OUT SPECIFICALLY AND
THE NIH DOCUMENTED THIS AND
INCLUDED US AND IT'S A WONDERFUL
THING AND I THANK THE NIH FOR
THAT.
I WANT TO START BY REFERENCING
AN ANCESTOR WHO DIED IN 1970
BEFORE I WAS BORN AND A
GRANDFATHER DIED AT AGE 70 AND
AS A BRIEF REFLECTION ON WHAT
HIS QUALITY OF LIFE MUST HAVE
BEEN MY MOM WHEN TALKING ABOUT
HIM AND REMEMBERING WHAT IT WAS
LIKE WHEN HE WAS SICK HIM
REMEMBERING HIM SITTING ON THE
COUCH AND NOT PART OF THE
CONVERSATION AND THINK WHAT IT
MUST BE LIKE FOR HIM AND MANY
PEOPLE SUFFERING WITH ALS BEFORE
THE LAST 20, 30 YEARS.
>>SO THE NEXT PERSON IN MY
DIRECT FAMILY WHO HAD ALS WAS MY
GRANDMOTHER LILLIAN PICTURED
HERE WITH MY MOTHER AND
GRANDFATHER.
SHE WAS DIAGNOSED IN THE LATE
1980s AND PASSED IN 1990 AND SHE
WAS 70 AND I WAS 7.
LILLIAN HAD AAN AUNT TELL ME
ABOUT THIS MASSIVE POWER CHAIR
AND A HADN'T SEEN HER IN IT
BEFORE AND WAS SCARED OF HER.
SHE WAS TRYING TO GIVE ME CANDY
OR SOMETHING AND I WAS FREAKED
OUT.
BUT APPARENTLY SHE HAD TROUBLE
USING IT ANYWAY PER MY AUNTS AND
UNCLES.
MY GRANDFATHER PICTURED HERE
WORKED HARD TO MAKE THEIR VERY
WHEELCHAIR UNFRIENDLY HOME
ACCESSIBLE WITH VERY LARGE
RAMPS.
THE INSTALLED AN ELEVATOR IN
THEIR HOME.
I DO KNOW THAT THEIR
NEIGHBORHOOD WAS NOT ACCESSIBLE.
IT WAS JUST A BUNCH OF CRAFT
SLATE SIDEWALK THINGS THAT NO
WHEELCHAIR WOULD BE ABLE TO
NAVIGATE AND I CAN'T IMAGINE SHE
WAS ABLE TO REALLY USE HER POWER
CHAIR OUTSIDE.
SO MY MOM CATHY WHO WAS VERY
WORRIED ABOUT ALS OVER THE YEARS
AND HAD DISABILITY BEFORE SHE
GOT A DIAGNOSIS DESPITE
ADVOCATING THAT FOR SELF WAS
DIAGNOSED IN 2017 AND PASSED
QUICKLY.
SHE MOVED TO A WHEELCHAIR
FRIENDLY APARTMENT.
I DON'T KNOW IF HER MOM'S
EXPERIENCE HIGHLIGHTED THAT WE
HAD AN UNFRIENDLY WHEELCHAIR
FRIENDLY HOME.
AND IT WAS IN A SOMEWHAT
WHEELCHAIR FRIENDLY NEIGHBORHOOD
WHEN SHE FINALLY STOPPED WALKING
AND GOT HER POWER CHAIR SHE
STARTED TO USE IT.
YOU CAN SEE PHOTOS HERE.
THE PHOTO ON THE LEFT -- THEY'RE
STARTING THE VIDEO.
I THINK THAT WAS ONE OF THE
FIRST TIMES SHE USED THE POWER
CHAIR AND YOU CAN SEE IN SOME
OTHER PHOTOS, THIS PHOTO I TOOK
WITH HER ON THE LEFT AT A
DOCTOR'S APPOINTMENT IN HER NEW
NEIGHBORHOOD AND BEFORE THE
APPOINTMENT A FEW DAYS BEFORE WE
WALKED THERE TO SEE IF WE COULD
MAKE IT WITHOUT A CAR AND WE
DID.
SHE WAS VERY EXCITED AND PROUD.
ANOTHER PHOTO THERE, MY MOM
LOVED SHOPPING AND SHE COULD NOT
SHOP OR DID NOT SHOP AFTER SHE
WAS DIAGNOSED AND THAT DAY I WAS
ABLE TO GET HER TO GO TO CVS
AROUND THE CORNER AND SHE
ENJOYED LOOKING THROUGH THE
AISLES ON THAT TRIP.
I WAS HAPPY WE WERE ABLE TO DO
THAT.
THAT'S PROBABLY THE LAST TIME
SHE WAS ABLE TO GO TO THE STORE.
SHE DID USE THE POWER CHAIR AND
DID DIE ONLY 10 MONTHS AFTER
GETTING IT.
SO JUST VERY SIMPLY, YOU MIGHT
SEE IN THE PROGRESSION OF THE
CIRCUMSTANCES OF MY GREAT
GRANDFATHER AND GRANDMOTHER THAT
TECHNOLOGY AND CIRCUMSTANCES CAN
HAVE PROFOUND AFFECTS ON PEOPLE
LIVING WITH ALS AND THEIR
CAREGIVER'S QUALITY OF LIFE.
THANK YOU VERY MUCH.
>>I'M GOING TO TALK ABOUT THE
THEMES WE CAME UP WITH AND THEN
ATTO PRIORITY.
QUALITY OF LIFE IS A CRITICAL
CONCERN WITH PEOPLE LIVING WITH
ALS AND CAREGIVERS.
WHEN WE REVIEWED THREE THINGS
POPPED OUT, MANAGEMENT OF THE
SYSTEMS AND HOW TO OPTIMIZE
DAILY LIVING AND COMMUNICATION
AND MOBILITY AND PROVIDING
EDUCATION AND DEVELOPMENT OF
RESOURCES AND TO HELP PEOPLE
IDENTIFY AND SHARE THE
RESOURCES.
WE ALSO WANTED TO RECOGNIZE
THERE ARE HEALTH CARE
DISPARITIES IN ALS TO DIRECTLY
IMPACT QUALITY OF LIFE.
AND EVERY INDIVIDUAL IS GOING TO
BE DIFFERENT IN HOW THE MIX
RESULTS IN THEIR QUALITY OF
LIFE.
THERE'S PSYCHOLOGICAL FACTORS.
THERE'S EXISTENTIAL FACTORS,
FINDING THE MEANING OF LIFE,
SPIRITUAL.
THERE'S EMOTIONAL SUPPORT AND
THERE'S RESOURCES AVAILABLE LIKE
JEAN EXEMPLIFIED JUST NOW IN
SHOWING THE POWER WHEELCHAIR AND
WHAT DIFFERENCE IT MADE IN HER
MOTHER'S MOBILITY AND ABILITY TO
GET AROUND AND THE OTHER POINT
IS THE QUALITY OF LIFE SHOULDN'T
BE ASSESSED BY THOSE AROUND BUT
BY PEOPLE LIVING WITH ALS
THEMSELVES.
OFTEN IF WE'RE IN GOOD HEALTH
WE'LL ESTIMATE THE QUALITY OF
LIFE AS PEOPLE LIVING WITH A
LIFE-THREATENING ILLNESS LIKE
ALS.
IT'S ALSO IMPORTANT TO RECOGNIZE
FOR PEOPLE WHO ARE LIVING WITH
ALS AND MAINTAIN A HIGH QUALITY
OF LIFE OVER TIME, THERE'S A
RESPONSE SHIFT IN WHAT BECOMES
IMPORTANT TO THEIR QUALITY OF
LIFE.
SO THE FACTORS THAT WERE
CRITICAL TO QUALITY OF LIFE MAY
EVOLVE FROM THOSE DEPENDENT ON
PHYSICAL FUNCTION LIKE
EXERCISING AND SO FORTH TO THOSE
THAT AREN'T MORE INTERPERSONAL
RELATIONSHIPS OR EXISTENTIAL
FACTORS AND SO THAT'S IMPORTANT
FOR EVERYONE TO RECOGNIZE.
AND WITH METRICS.
THERE'S NO CONSENSUS ON THE BEST
QUALITY OF LIFE METRICS.
THE REAL CHOICE IS DEPENDENT ON
WHAT YOU'RE LOOKING AT AND THE
SETTING YOU'RE MEASURING AND
WHAT YOU'RE STUDYING.
SO IT'S NOT A SINGLE SIMPLE
CONCEPT BUT REALLY YOU CAN
DEFINE IT AS HEALTH RELATED
WHICH REFLECTS YOUR HEALTH AND
DEPENDENT ON YOUR PHYSICAL WELL
BEING AND PHYSICAL FUNCTION OR
GLOBAL SCALES WHICH MORE RELY ON
PSYCHOLOGICAL EXISTENTIAL AND
THOSE SUPPORT FACTORS.
THEY'RE RELATIVELY INDEPENDENT
OF PHYSICAL FUNCTION.
SO WITHIN THESE CATEGORIES,
HEALTH RELATE AND GLOBAL THERE'S
INSTRUMENTS SPECIFIC FOR ALS OR
MORE GENERIC AND ALS SPECIFIC
INSTRUMENTS WOULD COMMONLY HAVE
SYMPTOMS THAT ARE PRESENT IN ALS
LIKE DROOLING AND TROUBLE
SPEAKING OR SWALLOWING OR MUSS
CRAMPS AND WOULD HAVE THINGS
MEASURED THAT ARE LITTLE OF
IMPORTANCE WHEREAS THE GENERIC
QUALITY OF LIFE HAS A BROADER
RANGE OF SYMPTOMS SOME OF WHICH
WOULD BE RELEVANT AND NOT.
IN CHOOSING A SCALE YOU HAVE TO
LOOK AT WHAT YOU'RE MEASURING
AND LOOKING AT AND WHAT TYPE OF
SCALE WOULD BE BETTER USED.
WITH THAT I'M GOING TO HAND IT
TO DR. PLOWMAN.
>>THANK YOU SO MUCH.
IT WAS A WONDERFUL EXPERIENCE
BEING ON THE WORKING GROUP AND
GLAD WE HAD JEAN AND LAYNE THAT
GAVE GOOD INFORMATION IN QUALITY
OF LIFE AS TERRY MENTIONED.
WE NEED TO GET IT.
MANY ARE FAMILIAR WITH THIS
MODEL THE INTERNATIONAL
CLASSIFICATION OF FUNCTIONING
DISABILITY AND HOW MORE COMMONLY
KNOWN AS THE ICF MODEL WHICH WAS
PUBLISHED TWO DECADES AGO NOW BY
THE WORLD HEALTH ORGANIZATION
AND THIS PSYCHOSOCIAL FRAMEWORK
DESCRIBES THE INTERACTIONS OF
INTERNAL AND EXTERNAL FACTORS TO
AN INDIVIDUAL HEALTH STATUS AND
HIGHLIGHTS NICELY THE INFLUENCE
OF THE ENVIRONMENT AND
ENVIRONMENTAL FACTORS LIKE
AVAILABLE PRODUCTS AND
TECHNOLOGY AND ENVIRONMENTAL
MODIFICATIONS THAT MAY BE MADE.
SUPPORT INFRASTRUCTURE, SOCIETAL
ATTITUDES, AVAILABLE SERVICES,
SYSTEMS AND POLICIES.
THESE ARE ALL THINGS WE HAVE THE
POWER TO MODIFY.
PERSONAL FACTORS CAN INCLUDE
DEMOGRAPHIC ATTRIBUTES, A
PERSON'S PREFERRED LIFESTYLE
HABITS, EDUCATION, SOCIAL
BACKGROUND, INDIVIDUALIZED
COOKING STYLE AND MOST
IMPORTANTLY THE INDIVIDUALIZED
PRIORITIES THAT CAN DIFFER
ACROSS PALS.
THIS UNDERSTANDS AND REMINDS US
QUALITY OF LIFE CANNOT BE
INFERRED OR ASSESSED BY A
CLINICIAN OR OTHERS BUT RATHER
TRULY DETERMINED BY AN
INDIVIDUAL WITH ALS OR CAREGIVER
AND THE QUALITY OF LIFE OF THE
CAREGIVER THEMSELVES.
AND ONE THING TO NOTE ON THE
MODEL IS QUALITY OF LIFE IS NOT
EXPLICITLY INCLUDED AT ALL IN
THIS FRAMEWORK.
IT WOULD HAVE BEEN INFERRED BUT
NOT INCLUDED.
NEXT, PLEASE.
ABOUT A DECADE LATER A MODEL WAS
CREATED FOR FUNCTIONING AND
QUALITY OF LIFE ARE INTERTWINED
AND CENTRAL CONCEPT TO CONSIDER
IN THE LIVES OF OUR PEOPLE THES
MODEL DEPICTS LIFE A COMPOSITE
OF LIFE, FUNCTION AND CONTEXTUAL
FACTORS AND AS APPRECIATED IN
THE INDIVIDUAL IT DEPICTS
QUALITY OF LIFE AS AN OUTCOME OF
THE INTERCONNECTED AND EVER
CHANGING INFLUENCES.
FINALLY AND I LOVE THIS MODEL
AND LATER THERE WAS A NESTED ICF
MODEL WITH INTERACTIVE NESTED
SUBSYSTEMS WITHIN THE OUTER MOST
SYSTEM WHICH IS NAMELY QUALITY
OF LIFE AND IT'S DEPICTED AS NON
LINEAR IN THE CONTINUUM MEANING
AT ANY GIVEN POINT IN TIME A
SYSTEM MAY LARGE OR SHRINK IN
TERMS OF THE INFLUENCE ON OVER
ALL QUALITY OF LIFE.
OUR WORKING GROUP UTILIZED THIS
TO OPTIMIZE QUALITY OF LIFE IN
PALS AND CALS AND DIFFERENT
INFLUENTIAL COMPONENTS TO
INCLUDE SYMPTOM MANAGEMENT
ACTIVITIES AND PARTICIPATION
WITH A FOCUS ON OUR FINDINGS
THAT COMMUNICATION AND MOBILITY
WERE DEEMED TO BE MOST IMPORTANT
IN SURVEY RESPONDENTS AND
FINALLY ENVIRONMENTAL SUPPORT.
I'LL OVERVIEW THE PRIORITIES.
UNDER SYMPTOM MANAGEMENT THERE'S
NEEDS TO BE AN UNDERSTANDING HOW
PHYSICAL, COGNITIVE AND
PSYCHOLOGICAL SYMPTOMS OF ALS
IMPACT QUALITY OF LIFE IN PALS
AND CALS.
WE WANT TO LOOK AT STRATEGIES.
SYMPTOMS IMPACT QUALITY OF LIFE
AND THE MENTAL WELL BEING OF OF
NOT ONLY PALS BUT CALS.
HISTORICAL SYMPTOM MANAGEMENT
APPROACHES HAVE BEEN REACTIVE IN
NATURE WHERE THE ALS PROGRESSION
DECIDES THE DECISION MAKING SUCH
AS THE COUGH ASSIST WHEN IT
DROPS TO A THRESHOLD LEVEL AND
MAKES THEM THE VICTIM AND AS THE
MERCY OF WHAT IS GOING TO OCCUR
NEXT.
AND WE PROPOSE A PROACTIVE
APPROACH THAT EDUCATES, ENABLES
AND IMPORTANTLY EMPOWERS PALS
AND CALS TO MAKE INFORMED,
EDUCATED AND PROACTIVE DECISIONS
IN A PERSONALIZED AND
PATIENT-CENTRIC MODEL WE WOULD
RECOMMEND FOR STUDY TO OPTIMIZE
QUALITY OF LIFE.
WE RECOMMEND MANAGEMENT
APPROACHES AND WOULD LIKE TO
QUICKLY HIGHLIGHT EMERGING
EXAMPLES OF SUCH APPROACHES SO
WE CAN FOCUS ON SOME OF THE
EXCITING AND POSITIVE
ADVANCEMENTS WE HAVE ON THE
HOERZON.
HORIZON.
FIRST I'D LIKE TO HIGHLIGHT
BEHAVIORAL PROACTIVE EMERGING
TECHNOLOGY OR OUTCOME AND HERE I
HIGHLIGHT SOME DATA FROM MY LAB
LOOKING AT A PROACTIVE
BEHAVIORAL SYMPTOM MANAGEMENT
AND PERFORMED AS WELL AS OTHER
PROGRAMMATIC LINE OF RESEARCH
INVESTIGATING PROPHYLACTIC
BREATHING EXERCISES WITH THE
INTEND TO INCREASE BREATHING,
AIRWAY DEFENSE AND PHYSIO LOGIC
CAPACITY AND EARLY EFFECTIVE
PALS.
HERE'S A PAL IN MY LAB.
THIS INDIVIDUAL HAD A BULBAR
ONSET AND THE PEAK COUGH FLOW
WAS WELL BELOW THE AGE EXPECTED
LEVEL.
THIS IS OVER A TWO-YEAR PERIOD
AND YOU CAN NOTE HERE IN THIS
INDIVIDUAL THAT WE WERE ABLE TO
INCREASE HIS PHYSIOLOGIC
CAPACITY FOR BREATHING AND
AIRWAY PROTECTION.
THERE'S A ROLE OF EARLY
RESPIRATORY EXERCISES FOR
QUALITY OF LIFE.
MORE WORK IS NEEDED IN THIS AREA
BUT OFFERS PROMISE OF PHYSICAL
MANAGEMENT THAT ARE COMMON AND
DEVASTATING IN PALS.
AN EXAMPLE OF PROGRESS MADE IN
THE PHARMACOLOGIC SYMPTOM
MANAGEMENT IS HIGHLIGHTED HERE
WITH THE 2017 SMITH AND
COLLEAGUES ARTICLE.
DR. RICHARD SMITH AND COLLEAGUES
EXAMINE THE TRIAL FOR A NEW DRUG
NUEDEXTA IT'S AN FDA APPROVED
DRUG FOR PBA AND SOME PALS
STARTED USING THIS AND THERE
WERE REPORTS ANECDOTALLY THEIR
SPEECH AND SWALLOWING SEEMED TO
BE IMPROVING.
THERE WAS AN ELEGANT CROSSOVER
DESIGN WITH A WASHOUT PERIOD AND
NOTED THAT WILL THERE'S A
SIGNIFICANT IMPROVEMENT ON
PATIENT REPORTED OUTCOME ON THE
BULBAR FUNCTIONAL SCALE AND IT
IMPROVED FOLLOWING A 28-DAY
TRIAL OF NUEDEXTA.
THIS IS THE FIRST TIME THE DATA
IS BEING PRESENTED.
AND THIS UNDER SCORES THE
IMPORTANCE OF SMALL PILOT
FUNDING.
WE GOT A $170,000 GRANT AND
PERFORMED A STUDY LOOKING AT AN
OPEN LABEL SMALL PILOT COHORT OF
30 INDIVIDUALS THE IMPACT OF A
30-DAY TRIAL OF NUEDEXTA AND
THERE'S A PLATFORM PRESENTATION
NEXT WEEK AND YOU GET A SNEAK
PEEK AT HERE AND THERE WAS A
SIGNIFICANT IMPROVEMENT IN THE
ALS FUNCTIONAL RATING SCALE
BULBAR SUB SCALE ON AVERAGE
IMPROVED BY ABOUT ONE PINT OVER
THE 30-DAY COURSE.
WHEN WE BROKE IT DOWN, 75% OF
PARTICIPANTS SIGNIFICANTLY
IMPROVED AND THEN 4.5%
MAINTAINED A 12% SCORE OR GOT
WORSE AND POSITIVE RESULTS AND
WE LOOKED AT PHYSIOLOGIC
OUTCOMES AND NOTED SECONDARY
OUTCOMES OF VIDEO FLEUR OSSCOPY
AND THIS INCREASED TO 29%
FOLLOWING THE PHARMACOLOGIC
INTERVENTION.
AND THEN FINALLY THERE WAS A
SPEECH OUTCOME MEASURE THAT
SIGNIFICANTLY IMPROVED OVER THE
COURSE OF THE TREATMENT.
OUR WORKING GROUP RECOMMENDED
THERE'S A CRITICAL NEED FOR THE
DEVELOPMENT OF BEST PRACTICES
FOR SCREENING AND ASSESSMENT IN
THIS AREA AS WELL AS NEEDED
MANAGEMENT GUIDELINES.
MY FINAL SLIDE WE RECOGNIZED
THERE'S LARGE CAPS IN THE
ASSESSMENT AND MANAGEMENT OF
MENTAL HEALTH OF PEOPLE LIVING
WITH ALS THAT NEED TO BE
EXPLORED.
AND THAT SPECIFICALLY ADVANCED
CARE PLANNING AND END OF LIFE
ARE NEEDED GREATLY FOR FUTURE
RESEARCH AND HERE I HIGHLIGHT A
BUCHL ARTICLE THAT WAS -- A
BEAUTIFUL ARTICLE THAT WAS
RECENTLY PUBLISHED I CAN REFER
YOU TO.
>>I'M MELANIE FRIED-OKEN AND
TALK ABOUT MAXIMIZING FUNCTION
AND ACTIVITIES AND
PARTICIPATION.
FIRST, IT'S CRITICAL WE CONDUCT
RESEARCH TO IDENTIFY
INTERVENTIONS THAT MAINTAIN
OPTIMAL FUNCTION ACTIVITIES OF
DAILY LIVING AND PARTICIPATION
THROUGHOUT THE COURSE OF
NEURODEGENERATION.
SECOND, THAT WE ESTABLISH
RESEARCH TO DEVELOP AND EVALUATE
SMART, DURABLE MEDICAL EQUIPMENT
AND NOVEL TECHNOLOGIES AND THIRD
THAT WE ESTABLISH RESEARCH TO
PROPOSE MEANINGFUL METRICS THAT
MEASURE THE AFFECT OF
INTERVENTIONS ON QUALITY OF
LIFE.
LET'S TALK ABOUT EACH ONE.
FIRST WE TO DESIGN AND INNERVATE
AND IT MUST BE BASED ON NEEDS S
AND THEY NEED TO CONTINUOUSLY
MONITOR CHANGES IN THE
ENVIRONMENT AND ACTIVITIES AND
IN IMPAIRMENT LEVELS.
SOME OF THESE CAN BE DONE
TECHNOLOGICALLY SO THERE'S A LOT
BEING DONE WITH SENSORS RIGHT
NOW THAT SHOULD BE INCLUDED
ANYONE ALS ASSESSMENT.
HERE'S SOME OF THE AREAS WE FIND
CRITICAL FOR INTERVENTIONS IN
THE AREA OF MOBILITY,
ENVIRONMENTAL CONTROL,
CONTEXT-DEPENDENT COMMUNICATION
SO BOTH FACE TO FACE AND REMOTE,
SPEECH AND WRITING AND WE ALL
KNOW HOW MUCH CONTEXT-DEPENDENT
COMMUNICATION HAS CHANGED FOR
ALL OF US WITH TEXTING, E-MAIL
AND NOT ONLY SITTING IN FRONT OF
A COMPUTER.
AND AGAIN, FUNCTIONS SUCH AS
SPEECH, SWALLOWING, BREATHING
AND AIRWAY CLEARANCE AS WELL AS
ACTIVITIES OF DAILY LIVING.
SO WE NEED TO COME UP WITH
INNOVATIVE INTERVENTIONS THAT
ASSESS EACH ONE OF THESE
CONTINUOUSLY OVER TIME.
ALL INTERVENTIONS WHATEVER WE
CHOOSE TO ASSESS AND AFFECT MUST
BE GUIDED BY PALS AND CALS
WITHIN THEIR COMMUNITIES FOR
ACCESSIBLE, INCLUSIVE AND
EQUITABLE USABLE-CENTERED DESIGN
AND I'LL USE THE TERM OVER AND
OVER AND DOING IT IN A
PHYSICIAN'S OFFICE IS IMPORTANT
BUT HAVING SOME WAY TO ASSESS
AND IT WILL AFFECT QUAL OF LIFE
MOST.
AND THE SECOND WAS TO DEVELOP
AND TEST NOVEL AND SMART
ASSISTIVE TECHNOLOGIES.
AND MACHINE THEIR FUNCTIONAL
AFFECTS ON QUALITY OF LIFE AND
SMART HOME TECHNOLOGIES
AVAILABLE FOR ALL OF US AND
WEARABLE TECHNOLOGIES WE CAN USE
FOR MONITORING.
SO MANY OF US HAVE CHOSEN TO USE
ALEXA, FOR EXAMPLE, FOR
ENVIRONMENTAL MANAGEMENT AND
ADDING THAT TO THE TOOLBOX OF
ASSISTIVE TECHNOLOGIES FOR PALS
IS CRITICAL.
A LOT OF THESE THINGS ARE
AVAILABLE ON THE MARKET AND WHAT
WE MUST DO IS IDENTIFY THE ITEM
AND THEN MOST IMPORTANTLY FORM
AN INTERVENTION OR TRAIN PEOPLE
ON HOW TO USE IT.
JUST HANDING SOMEONE AN
ASSISTIVE TECHNOLOGY ISN'T GOING
TO DO IT.
WE NEED TO HAVE TREATMENT PLANS
AND EXPERTS WHO CAN HELP
IMPLEMENT IN THE COMMUNITY.
WE NEED TO DESIGN SPECIFIC
INTERVENTIONS AND ASSISTIVE
TECHNOLOGIES THAT EXTEND
FUNCTION IN NOVEL WAYS.
THIS GOES BACK TO WHAT JEAN
STARTED US OFF WITH.
SO FOR OUR NEW TECHNOLOGIES OR
DURABLE MEDICAL EQUIPMENT
RESEARCH AND DEVELOPMENT, WE
NEED TO HAVE AT LEAST THREE
FUNDAMENTAL DATA SETS THAT ARE
ACQUIRED OVER TIME.
THESE ARE IN USER PERFORMANCE.
SO HOW DOES THE USER INCREASE OR
WHAT ARE THE CHALLENGES OVER
TIME FROM NEURODEGENERATION.
THE DEVICE PERFORMANCE AS WELL
AS ADAPTATION AND CUSTOMIZATION
THROUGH MACHINE LEARNING.
WHAT DOES THAT MEAN?
IT MEANS AS THE PERSON'S
FUNCTIONAL SKILLS CHANGE THE
TECHNOLOGY SHOULD AUTOMATICALLY
ADJUST AND CHANGE WITH THEM.
LET ME GIVE YOU SOME EXAMPLES.
I RECENTLY GOT A NEW CAR BECAUSE
I NEED THE ASSISTIVE DRIVING
FEATURES IN THE CAR.
THOSE SHOULD BE INCLUDED IN
EVERY WHEELCHAIR.
SO IF HOME IS WHERE A PAL SPENDS
THE MAJORITY OF THE TIME THE
POWER MOBILITY SHOULD BE
FAMILIAR WITH THE ENTIRE PLAN OF
THE HOME.
SHOULD KNOW WHEN YOU'RE GOING TO
NEED TO MOVE RIGHT OR LEFT.
CONTEXT-DEPENDENT LEARNING IS
CRITICAL FOR WHEELCHAIRS.
AND FOR COMMUNICATION DEVICES,
WE NEED TO KNOW ALL THE ACCESS
METHODS.
THE DEVICE SHOULD LEARN IF
SOMEONE IS TYPING, WHEN THEY'RE
MAKING ENOUGH MISTAKES TO SAY
IT'S TIME TO MOVE TO A DIFFERENT
ACCESS METHOD.
MAYBE WE MOVE FROM MOVING ON A
KEYBOARD TO CHOOSING A MOUSE OR
JOY STICK AND MOVE THERE TO A
HEAD MOUSE OR EYE GAZE
TECHNOLOGY AND THE TECHNOLOGY
SHOULD HELP IDENTIFY WHEN WE
NEED TO MOVE TO THAT STATE AND
HAVE A MECHANISM TO TEACH US HOW
TO USE THE NEXT ACCESS.
THAT'S WHAT I MEAN BY MAKING
SOMETHING SMART AND DETERMINE
HOW'S THE USER DOING AND WHAT'S
THE DEVICE NEED TO DO AND HOW
CAN THE DEVICE BE CUSTOMIZED AND
ADAPTED BASED ON WHAT THE PERSON
DOES?
THIS IS REALLY GOING TO AFFECT
FOUR THINGS.
IT'S GOING TO HELP AND INCREASE
ADOPTION BY PALS.
IT'S GOING TO DECREASE
ABANDONMENT BY PALS.
IT'S GOING TO INCREASE
PARTICIPATION WHICH IS OUR MAJOR
GOAL FOR QUALITY OF LIFE AND
FINALLY, IT'S GOING REDUCE SOME
OF THE CAREGIVER
RESPONSIBILITIES BECAUSE THE
TECHNOLOGY CAN TAKE THAT OVER
AND THE CAREGIVERS CAN BE
DEVOTED TO WORKING WITH THE PALS
INSTEAD OF FIGURING OUT HOW TO
USE THAT DEVICE.
THEN OUR THIRD FOR FUNCTION
ALLIES RESEARCH AND WE NEED
METRICS AND THESE INCLUDE EASE
AND USE AND IMPLEMENTATION OF
ASSIST OF A TECHNOLOGIES AND
WILL DEPEND ON INDIVIDUALS AND
THEIR COMFORT LEVEL.
FLEXIBILITY.
AND BY THAT I MEAN THE ASSISTIVE
TECHNOLOGIES NEED TO CHANGE OVER
TIME SHORT AND LONG-TERM
EFFECTIVENESS FOR DAILY
PARTICIPATION AND THAT MEANS IN
DIFFERENT CONTEXTS WITH
DIFFERENT PEOPLE.
WE NEED TO FIND A WAY TO HAVE
RESULT REDUCTION OF CAREGIVER
RESPONSIBILITIES THROUGH THE
ASSISTIVE TECHNOLOGIES SO WE CAN
MAXIMIZE ACTIVITIES AND
PARTICIPATION AND THAT SHOULD BE
THE GOAL OF THE CARE PROVIDERS.
WE NEED TO INCLUDE USER
EXPERIENCE AND SATISFACTION
WITHIN INTERVENTIONS AND
ASSISTIVE TECHNOLOGIES AND
FINALLY BY PATIENT-REPORTED
OUTCOME MEASURES TO INFLUENCE
EVERYTHING RELATED TO QUALITY OF
LIFE.
I WANTED TO MAKE TWO ADDITIONAL
POINTS.
YESTERDAY THE TRANSLATIONAL
RESEARCH GROUP TALKED ABOUT
LOOKING AT GENES AND PROTEINS.
I'D LIKE TO MAINTAIN ASSISTIVE
TECHNOLOGY IS A TRANSLATIONAL
ACTIVITY.
THE BRAIN COMPUTER INTERFACE AND
INSTEAD OF MOVING FROM TEST TUBE
WE MOVE FROM BRAIN SIGNALS AND
THEY NEED TO BE TRANSLATED INTO
FUNCTION AND INTENT.
THE SAME DISCUSSION HELD
YESTERDAY ABOUT GENES AND
PROTEINS CAN BE HELD TODAY AS
WELL WITH DIFFERENT ASPECTS OF
ASSISTIVE TECHNOLOGY.
THOSE WHO WORK IN TRYING TO
MAXIMIZE FUNCTION AND ARTS
PASSION THROUGH ASSISTIVE
TECHNOLOGY, WE OFTEN COME UP
WITH THE PROOF OF CONCEPT AND
PROOF OF PRODUCT AND THEN
PARTNER WITH INDUSTRY TO MOVE
THOSE PRODUCTS FORWARD.
LIKE THEY SAID YESTERDAY, EARLY
DRUG DISCOVERY MAY 10 TO 15
YEARS TO BRING TO MARKET.
SOME THINGS WE'RE WORKING ON FOR
MAXIMIZING ACTIVITIES AND
PARTICIPATION WILL TAKE AS LONG
AS WELL.
THE THING ABOUT MAXIMIZING
FUNCTION ACTIVITIES AND
PARTICIPATION IS WE CAN'T DO IT
WITHOUT PATIENT-REPORTED
OUTCOMES AND PARTICIPATORY
ACTION RESEARCH FROM THE PALS
COMMUNITY.
THANK YOU.
>>I'M GOING TO BE INTRODUCING
PRIORITY THREE WHICH IS
EVIDENCE-BASED EDUCATIONAL
RESOURCES AND BEFORE WE JUMP
INTO OUR BULLETS WELL START WITH
ANOTHER BEAUTIFUL ANECDOTE FROM
JEAN.
>>THERE'S SO MANY THINGS MY MOM
AND FAMILY MAYBE DID WRONG OR
DAD THAT COULD HAVE MADE HER
LIFE BETTER HAD WE DONE WHAT
EVIDENCE AND PRIOR EXPERIENCES
OF PEOPLE WOULD HAVE SUGGESTED.
THE HAD THE POWER CHAIR YOU SAW
AND ONE THING THAT THESE NEW
POWER CHAIRS CAN DO OFTEN IS LAY
FLAT AND ELEVATE SO THAT A
PERSON WHO CANNOT TRANSFER
THEMSELVES COULD BE EASILY
TRANSFERRED IT ANOTHER FLAT
SURFACE.
I REMEMBER HAVING A LOT OF
TROUBLE TRANSFERRING MY MOM.
WE HAD PAID AIDS THAT HANDLED
MOST THE TRANSFERS FOR MY MOM
AND WHO SHE WANTED TO DO IT BUT
UNDER CIRCUMSTANCES MY HUSBAND
AND I HAD TO HELP TRANSFER HER
AND WE HAD A LOT OF TROUBLE.
I WAS SEEING COMMENTS ABOUT
USING THE POWER CHAIRS IN THAT
WAY TO RECLINE AND WE COULMY MO
RECLINER CHAIR THAT ALSO LAID
FLAT AND SHE HAD HER BED WE
COULD HAVE DONE THAT WITH BUT WE
DIDN'T DO IT AND I WAS THINKING,
JUST HAVING SOME KIND OF PRIMER
THAT WAS EASILY ACCESSIBLE AT
ALL TIMES WHENEVER THAT HAPPENED
IN THE MIDDLE OF A SATURDAY AND
WE NEEDED TO TRANSFER HER, WE
COULD HAVE CONSULTED THAT AND
REALIZED THAT.
I WANTED TO ADD ANOTHER FLAVOR
TO THAT WAS I DID KNOW THAT AND
ONE OF THE FIRST TIMES I WAS
TRYING TO TRANSFER HER GETTING
SURGERY WITH A FEEDING TUBE IN
THE HOSPITAL WE WENT BACK TO THE
SURGERY CENTER BEFORE SHE WAS
GOING GET BROUGHT IN AND I WAS
GETTING PREPARED LIKE OKAY, MOM,
WE'RE GOING TO RECLINE THE CHAIR
AND ELEVATE IT AND I'LL HELP YOU
MAKE A TRANSPORTER AND WE'LL
MOVE YOU AND OF COURSE HOSPITALS
ARE BUSY PLACES AND TWO
TRANSPORTERS COME IN AND QUICKLY
CHUCK HER UP ON A BLANKET AND
TRANSFER HER AND MY MOM WASN'T
PREPARED AND WASN'T RELAXED.
I FEEL THAT PROBABLY TO DO IT
BEST SOMEONE NEEDS TO BE RELAXED
NOT TENSE.
MY MOM'S EXPERIENCE WAS I NEVER
WANT TO DO IT THIS WAY AGAIN AND
WHEN YOUR MOM TELLS YOU
SOMETHING LIKE THAT YOU, DON'T
WANT TO GO AGAINST IT SO I PUT
IT OUT OF MY MIND WE'D NEVER TRY
THAT AGAIN THOUGH WE HAD THIS
WONDERFUL PIECE OF EQUIPMENT WE
WOULD HAVE BEEN USING IN THE
HOME.
PERHAPS HAVING A RESOURCE THAT
IS READABLE WOULD HAVE MADE
USING THAT BETTER GOING FORWARD.
THANK YOU SO MUCH.
>>SO IN THAT VEIN, WE WANT TO
IDENTIFY RESOURCES AND USING
PROFESSIONALS AND RESEARCHERS
AND WE ALSO WANT TO USE THE
EXPERTISE OF PERSONS WITH ALS
AND CAREGIVERS INCLUDING PAID
CAREGIVERS BECAUSE WE KNOW
OTHERS HAVE EXPERTISE FROM OTHER
GROUPS.
WE ARE ASKING FOR CRATING AND
DEVELOPING THE LIST IN
CONJUNCTION WITH ALL THE PLAYERS
TO DEVELOP THE BEST SET OF
PRACTICES AND RESOURCE.
NEXT WE'RE HOPING TO IMPROVE
ACCESS TO EDUCATION AND
RESOURCES ABOUT ALS TO ALL THOSE
IMPACTED BY ALS.
THE EMPHASIS IS ALL THOSE
IMPACTED BY ALS.
ONE OF THE PIECES OUR PRIORITY
GROUP WITHIN THIS WORKING GROUP
THOUGHT THROUGH WAS THE FACT
THAT WE KNOW EVERYONE THAT IS
BEING IMPACTED BY ALS IS NOT
BEING IDENTIFIED.
AND HOW DO WE ENSURE THE
EDUCATION IS GETTING TO OTHER
SPACES OTHER THAN THE
TRADITIONAL ALS CLINIC OR ALSO
RESEARCH SPACES WHERE PEOPLE GO
SEEKING FOR CURES.
WE'RE HOPING IT WILL DISSEMINATE
THE BEST PRACTICES AND RESOURCE
FOR CAREGIVERS AND PERSONS WITH
ALS AND CLINICIANS, ALLIED,
PROFESSIONALS AND RESEARCHERS
AND WE KNOW SIMILAR TO THE FIRST
PRIORITY IT WILL TAKE PLAYERS IN
CONJUNCTION WITH ONE ANOTHER AND
US PUSHING BEYOND THE BOUNDS OF
OUR TRADITIONAL ALS BASIS TO
MAKE SURE WE'RE IDENTIFYING ALL
THOSE IMPACTED BY ALS.
FINALLY, OUR THIRD LARGER
SUBBULLET FOR EDUCATIONAL
RESOURCES IS OF THE COMPLIED
INFORMATION, WE WANT TO ADDRESS
ALL THE ASPECTS OF CARE FOR
PERSONS WITH ALS AND ALSO BE
EXPLICIT ABOUT ENGAGING A
DIVERSE GROUP OF PEOPLE IMPACTED
BY ALS AS MENTIONED IN THE
BEGINNING WE KNOW THERE ARE
HEALTH CARE AND HEALTH
DISPARITIES WITHIN THE ALS SPACE
THAT HAS A LOT TO DO WITH TIME
TO DIAGNOSIS AND BEING ABLE TO
ACCESS A CLINIC AND BIASES
WITHIN CLINICAL SPACES AND
EXISTING ISSUES.
IN THE UNITED STATES THAT
IMPACTS HOW WE UNDERSTAND CARE
AS A CAPITALIST INDUSTRY.
BECAUSE OF THAT AND BY COMPILING
THE INFORMATION WE'RE HOPING TO
ENGAGE MULTI-DISCIPLINARY CARE
PRACTICES ABOUT PROMPT DIAGNOSIS
AND BEST CARE PRACTICES FOR ALL
ALS CLINICS AND CREATE
STANDARDIZATION AROUND THAT AND
BE SPECIFIC TO PERSONS WITH ALS
AND CAREGIVERS OF ALS ABOUT
THEIR PERSONAL BEST CARE
PRACTICES EITHER AROUND
NUTRITION OR SELF-CARE, ABOUT
HOW TO FIND JOY AND HAPPINESS IN
LIFE WHILE LIVING WITH A
TERMINAL DISEASE.
WE WANT IT TO BE RESOURCES WE
COMPILE.
WE WANT TO ENSURE THERE'S ACCESS
INFORMATION RELATIVE TO
PROVIDERS AND PERSONS WITH ALS.
ONE OF THE THINGS I THINK IS
VALUABLE ABOUT THE SPACE IS WE
ARE SMASHING THE HIERARCHY OF
SIMPLY THE PHYSICIAN OR
PHYSICIAN SCIENTIST BEING THE
ONLY EXPERT IN THE ALS SPACE AND
REALLY VALUING THAT AND HEALTH
CARE PROVIDERS NEED RESOURCES TO
HELP SUPPORT THEM AND HOW THEY
SHARE INFORMATION TO PATIENTS AS
WELL.
WE'RE ALSO GOING TO ENSURE WE
HOPE TO ENSURE THIS IS AN
INCLUSIVE REVIEW TO ENSURE
HEALTH EQUITY.
AND IF WE ARE NOT ACCESSING AND
IDENTIFYING ALL PERSONS WITH ALS
THEN IT'S INCUMBENT UPON US AS
PEOPLE ADVOCATES IN THE
COMMUNITY TO ENSURE THAT OUR
REVIEW IS AT HEARING TO HEALTH
EQUITY STANDARDS AND MAKING SURE
EVERYONE IS BEING ACCESSED.
SO IN THAT VEIN WE WANT TO
EMPHASIZE THAT PEOPLE FROM
HISTORICALLY UNDER REPRESENTED
BACKGROUNDS ARE BEING SUPPORTED
IN THESE TYPES OF RESOURCES.
THAT SPECIFICALLY INDIVIDUALS
AND RURAL COMMUNITIES AND
INDIVIDUALS WHERE ENGLISH IS A
SECOND LANGUAGE AND NON-MALE
INDIVIDUALS, PERSONS WITH ALS
THAT ARE YOUNGER.
SO YOUNGER THAN 45, 50, THAT
TYPICAL ALS AGE RANGE WHY WE'RE
SEEING OUR POPULATION IN THE
CLINIC AND WHAT MY RESEARCH HAS
FOCUSSED ON OVER THE LAST DECADE
IS ENSURING THAT BLACK,
INDIGENOUS AND PEOPLE OF COLOR
ARE GETTING THE RESOURCES NOT
ONLY ABOUT WHAT ALS IS AS A
DISEASE BUT ALSO ENSURING THAT
ONCE THEY ARE DIAGNOSED AND ARE
IN OUR ALS COMMUNITY THAT
THEY'RE RECEIVING THE
INFORMATION FOR BOTH SUPPORTING
THEIR CAREGIVERS AND SUPPORTING
THEMSELVES IN THE BEST WAYS
POSSIBLE.
AND FINALLY, WE'RE HOPING
RESOURCE, SUGGESTIONS AND BEST S
WILL ALWAYS ADDRESS THE SOCIAL,
CULTURAL AND ETHNIC AND ECONOMIC
BARRIERS THAT MAY EXIST IN
ACCESSING AND USING THE
RESOURCE.
IT'S IMPORTANT TO OVEREMPHASIZE
THIS POINT BECAUSE A LOT OF THE
RESOURCES AND TECHNOLOGIES THAT
WE KNOW ABOUT ALS ARE NOT ALWAYS
ACCESSIBLE GIVEN A BACKGROUND.
BECAUSE OF THAT WE WANT TO
ENSURE OUR EVIDENCE-BASED
EDUCATIONAL RESOURCES ARE ABLE
TO BE UTILIZED AND ACCESSED BY
THESE VARIOUS GROUPS AND AS
WE'RE COMPILING THIS INFORMATION
WE HAVE NOT ONLY A HEALTH EQUITY
LENS IN THINKING THROUGH THE
BARRIERS BUT ECONOMIC BARRIERS.
AND I'LL REFLECT ON
EVIDENCE-BASED EDUCATIONAL
RESOURCES FROM THE CLINICIAN
PERSPECTIVE.
>>THERE ARE A FEW THINGS
HIGHLIGHT FROM THE CLINICIAN'S
PERSPECTIVE AN THIS SLIDE THIS
SUMMARIZES QUICKLY ABOUT THAT
FROM A CLINICIAN PERSPECTIVE A
LOT OF THE TOPICS THAT WAS
REVIEWED BY DR. CARTER EXIST IN
SOME WAY, SHAPES OR FORMS IN
PIECES.
ONE OF THE CHALLENGES IS FIRST
TO HAVE A WAY TO IMPROVE THE
EXISTING RESOURCES AND BY WAY OF
RESEARCH AND THEN TRYING TO
COMPILE THEM IN ONE SORT OF HUB
THAT COULD BE EASILY
DISSEMINATED BOTH ELECTRONICALLY
AND ALSO IN THE CLINICS AND SO
THAT WOULD BE THE EDUCATION
RESOURCE.
BOTH THAT IS USABLE FROM THE
CLINICIAN PERSPECTIVE AND FOR
PEOPLE LIVING THROUGH THE
DISEASE AND GOING THROUGH THE
JOURNEY WITH ALS.
THE SECOND I WANTED TO POINT OUT
WE COULDN'T SQUEEZE IN IS
EDUCATION TOWARDS THE HEALTH
CARE PROVIDERS THEMSELVES.
THE FIRST IS OBVIOUSLY TRYING TO
MAKE PROMPT DIAGNOSIS AND THE
ALS ASSOCIATION HAS CHAMPIONED
AN INITIATIVE TO HAVE PHYSICIANS
THINK ABOUT ALS AS SOON AS
POSSIBLE AND RAISING AWARENESS
SO THAT PEOPLE THAT ARE
SUSPECTED TO HAVE ALS COULD BE
REFERRED TO SPECIALTY CENTERS
QUICKER SO THE DIAGNOSIS COULD
BE MADE QUICKER.
A DELAY IN DIAGNOSIS HAS A BIG
IMPACT ON QUALITY OF LIVING WITH
THE DISEASE AND DELAYS TO
TREATMENT FOR SYMPTOM FUNCTION
AND MEDICATIONS.
THE OTHER ASPECT IS ALSO TO
EDUCATE HEALTH CARE PROVIDERS IN
COMMUNICATION.
THIS IS SOMETHING THAT EXISTS
ALREADY FOR OTHER DISEASE SPACES
BUT IN ALS PROVIDING THE
DIAGNOSIS AND HAVING THE
DISCUSSION IN A WAY THAT'S
SENSITIVE AND EMPATHETIC CAN
HELP IMPROV PEOPLE'S FIRST BRUSH
WITH THE WORDS MYOTROPIC LATERAL
SCLEROSIS AND HAVE AN IMPACT ON
THE WAY THE JOURNEY PROCEEDS
FORWARD THROUGH THE TRANSITIONS
OF SYMPTOMS AND AS THE DISEASE
PROGRESSES.
I THINK THAT COMMUNICATION,
EDUCATION IS IMPORTANT.
HOPEFULLY THAT CAN BE
ENCOMPASSED IN THE EDUCATION
COMPONENT.
WITH THAT I'D LIKE TO TURN IT
OVER TO STEVE FOR THE
PERSPECTIVE OF A PERSON LIVING
WITH ALS.
>>THANK YOU.
I'VE HAD MY SLIDE UP FOR A WHILE
SO YOU'VE SEEN THAT AND THIS IS
FROM A PALS' PERSPECTIVE.
AS WE HEARD, THE RESEARCH
COMMUNITY IS NOW IN A POSITION
TO MAKE THE REALIZATION OF ALS
AS A LIVABLE DISEASE A REALITY.
THE TREATMENTS AVAILABLE TODAY
SHOW PROMISE OF SLOWING THE
DISEASE AND WITH THAT HAVE
CAUTIONARY FLAG THAT COME TO MY
MIND AND HOPEFULLY YOU AS WELL
IN THAT MAKING ALS A MORE
LIVABLE DISEASE REQUIRES US TO
MAKE SURE WE PAY ATTENTION TO
THOSE FOLKS' QUALITY OF LIFE AS
THEY LIVE LONGER WITH ALS.
IT WOULD BE A TRAGEDY TO SEE
FOLKS LIVING LONGER WITH ALS BUT
A DIMINISHING QUALITY OF LIFE AS
THEY GO ALONG THAT CONTINUUM.
SO SOLUTIONS TO IMPROVE QUALITY
OF LIFE NEED FURTHER
UNDERSTANDING AND ANALYSIS AND
RESULT IN POSITIVE MEANINGFUL
SOLUTIONS FOR EVERYBODY.
I ALSO LOOKED AT THIS THROUGH
THE LENS OF ATTEND GREAT CLINIC
CARE IN BOSTON AND HOW QUALITY
OF LIFE IS LOOKED AT WHEN I
VISIT CLINIC TO SAY THAT WE
DON'T TALK ABOUT THIS A LOT IN
MY CLINICAL VISITS.
I THINK WHAT I'VE COME TO
REALIZE IS THAT THE ALS FRS
SCALE AND BREATHING SCORES
PERHAPS GIVE AN INDICATION OF
ONE'S QUALITY OF LIFE BUT
WITHOUT ASKING DIRECT QUESTIONS
THEY SOMETIMES CREATE
ASSUMPTIONS AND SO I THINK IT'S
IMPORTANT THAT WE INCORPORATE
THE THREE RECOMMENDED PRIORITY
AREAS TO TALK ABOUT AND
REIMAGINE STANDARD OF CARE AND
INFORM RESEARCH PARTNERS.
THE OTHER THING THAT DAWNED ON
ME YESTERDAY IN PARTICIPATING IN
THIS IS THAT A BETTER QUALITY OF
LIFE OF PATIENTS LIVING WITH
THIS DISEASE CAN EASILY
TRANSLATE TO MORE CLINICAL TRIAL
PARTICIPATION AND RETENTION.
SO THERE'S A BENEFIT TO THE
RESEARCH COMMUNITY AS WELL.
MELANIE TALKED ABOUT THIS AND
OTHERS AS WELL AND THERE'S BEEN
TECHNOLOGY IN QUALITY OF LIFE
AND IMAGINE IN LOU GHERIG WAS
ALIVE AND GOT TO LOOK AT WHAT'S
AVAILABLE I THINK HE'D BE
IMPRESSED WITH THE PROGRESS
WE'VE MADE.
THE SCALE OF INNOVATION NEEDS TO
CHANGE AND NEEDS TO BE FASTER
WITH BROADER IMPACT.
WE HEARD THE ALS CLOCK IS
TICKING AND WAS MENTIONED BY
SEVERAL PEOPLE.
ARC OF INNOVATION IS ALSO MOVING
PRETTY RAPIDLY AS WELL.
IN MY OBSERVATION AND THE WORK
I'VE DONE FOR ADVOCATING FOR ALS
IN THE FIVE YEARS IS WE TEND TO
BE AT THE BOTTOM OF THE CURVE IN
GENERAL AND NOT RIDE THIG CURVE.
HERE'S AN EXAMPLE OF HOW THINGS
ARE CHANGING RAPIDLY IN WAYS
THAT WE CAN TAKE LOOK AT
OPPORTUNITY WITHIN THE CHANGES.
WE HEAR ABOUT V.R.
IT'S A RAPIDLY EVOLVING
TECHNOLOGY AND AS WE STRIVE TO
WORK FOR A WORLD WITHOUT ALS
THERE WOULD VERY WELL BE A
VIRTUAL WORLD WHERE PEOPLE CAN
PARTICIPATE IN AN PARTICIPATE IN
WAYS YET TO BE IMAGINED.
AND ROBOTICS WHICH HASN'T BEEN
MENTIONED MUCH ARE NOT STAGNANT
EDEAVORS.
THEY'RE EVOLVING AND IMPROVING
AND PATIENTS LIKE ME NEED TO BE
AFFORDED THE OPPORTUNITIES TO
UTILIZE THEM.
WE WENT TO MAKE SURE NEW
TECHNOLOGIES ARE AFFORDABLE TO
ALL.
I LEAVE YOU WITH THE FINAL
THOUGHT AS I WATCH THE HEROIC OF
CHANGING THE COURSE OF AN
ASTEROID, I THOUGHT TO MYSELF,
GOSH, IMAGINE IF WE CAN DO THAT
WHAT WE CAN DO TO CHANGE AND
IMPROVE THE COURSE OF A PERSON
LIVING WITH ALS'S QUALITY OF
LIFE.
I TURN IT BACK TO TERRY AND JEAN
FOR I GUESS OUR Q&A SECTION.
>>HOW TO FOR PRESENTING OUR
WORK.
>>WE HAD QUESTIONS COME IN
AHEAD OF TIME AND I KNOW WE'VE
BEEN WORKING HARD TO COLLATE THE
QUESTIONS THAT HAVE BEEN JUST
ANSWERED IN THE CHAT.
FEEL FREE TO KEEP PLACING THEM
IN THERE IF YOU HAVE ANY.
FOR THE FIRST QUESTION I'M GOING
ADDRESS THIS TO TERRY.
AND HOW CAN WE DEVELOP A BETTER
DISEASE QUALITY OF LIFE MEASURE?
>>THIS QUESTION CAME IN EARLY
SO I HAD THE OPPORTUNITY TO
ACTUALLY ASK ZAK THE PERFECT
PERSON TO ANSWER THAT AND I'LL
READ BACK THE RESPONSE.
HE SAID I DON'T THINK WE NEED A
BETTER INSTRUMENT RATHER WHAT
WE'RE TRYING TO GAUGE IN A
PARTICULAR STUDY AND USE THE
INSTRUMENT THAT'S MOST SUITABLE.
FOR EXAMPLE, IF WE'RE USING A
MEDICATION THAT WE BELIEVE WILL
IMPROVE SLEEP, WE NEED TO ASSESS
THAT WITH A SLEEP-RELATED
QUALITY OF LIFE INSTRUMENT.
IF WE'RE USING ONE THAT IMPROVES
PAIN OR LOOKING AT PAIN, WE NEED
A PAIN-RELATED QUALITY OF LIFE
INSTRUMENT.
AND IF WE'RE TRYING TO JUDGE
OVER ALL AFFECTS ON HEALTH WE
SHOULD GET A QUALITY OF LIFE
INSTRUMENT.
AND LOOKING AT ALL ASPECTS OF
LIFE AND FOR RELATIONSHIPS AND
SUPPORT AND SPIRITUALITY AND
EXISTENTIAL FACTORS WE NEED A
SPECIFIC QUALITY OF LIFE.
IT'S ABOUT CHOOSING THE RIGHT
INSTRUMENT, KNOWING WHAT YOUR
QUESTION IS AND CHOOSING THE
RIGHT INSTRUMENT AND A
FUNDAMENTAL QUALITY OF ANY
INSTRUMENT WE USE IS IT SHOULD
BE SENSITIVE TO CHANGE FOR THE
MEASURE WE'RE HOPING TO IMPACT.
IT'S CHOOSING THE RIGHT
INSTRUMENT.
THANK YOU, GENE.
HOW CAN WE ENSURE BEING A
GENETIC CARRIER IS NOT A REASON
FOR LIFE INSURANCE COMPANIES TO
DENY LONG-TERM INSURANCE AND
JEAN, THIS ONE'S BACK AT YOU.
>>THIS IS AN IMPORTANT TOPIC.
AND THIS AN IMPORTANT REASON
BEFORE JUMPING INTO PREDICTIVE
GENETIC TESTING PEOPLE SPEAK TO
THE GENETIC COUNSELORS AND HAVE
THE ABILITY TO SPEAK WITH YOU
ABOUT ALL THIS STUFF TO THE
PRACTICALITIES OF THE QUESTION.
THAT'S A POLITICAL QUESTION
ABOUT WHAT LAWS EXIST.
INTERESTINGLY, FLORIDA OF ALL
STATES OUTLAWED GENETIC
DISCRIMINATION FOR LIFE
DISABILITY AND LONG-TERM CARE
INSURANCE IN 2020.
I HAVE NOT SEEN HEADWINDS OF
THAT GOING ANYWHERE ELSE BUT
COULD HAPPEN AND I KNOW WHEN THE
LAW WAS PASSED THAT PROTECTS
HEALTH INSURANCE AND EMPLOYMENT
AT COMPANIES LARGER THAN 15
PEOPLE BASED ON STATUS THE
SCIENTIFIC COMMUNITY PLAYED A
PART IN EDUCATING WHY THAT WAS
IMPORTANT AND I'M SURE WE CAN
LEAN ON THE NIH AND OTHER
SCIENTIFIC LEADERS TO MAKE THAT
CASE IF CONVERSATIONS ABOUT
EXPANDING THAT EVER GETS GOING.
THANK YOU VERY MUCH.
STEVE, IS INDUSTRY WORKING WITH
PEOPLE WITH ALS AND CLINICIANS
FOR SMART ASSISTIVE
TECHNOLOGIES?
>>THAT'S MY PASSION AND IT'S
BEEN MY GOAL SINCE DAY ONE.
I'VE MADE PROGRESS BUT IT IS
SLOW BUT I THINK INDUSTRY AND
HIGH TECH IN PARTICULAR HAS A
ROLE TO PLAY AND HELPING MAKE
THIS A REALITY.
ONE AREA I'VE WATCHED IS THE
AREA OF HOME AUTOMATION.
I CONSIDER MYSELF PRETTY TECH
SAVVY AND SOME OF THIS
CONNECTING TO HOME AUTOMATION
CAN BE A CHALLENGE.
OFTEN TIMES YOU DON'T KNOW WHAT
TO BUY.
SOMETIMES YOU PURCHASE THE WRONG
THING AND WHEN YOU HAVE THE
WRITE THING IT SIMPLY DOESN'T
WORK.
AN EXAMPLE OF INDUSTRY COMING
TOGETHER, I'VE BEEN FOLLOWING A
MUCH NEEDED APPROACH AND THAT'S
A STANDARD ALL MANUFACTURERS
WHETHER APPLE, AMAZON, GOOGLE,
MICROSOFT ALL FOLLOW ONE
STAND
STANDARD.
THAT SHOULD HELP SIMPLIFY THOSE
OF US WHO GET SOMETHING FROM
AMAZON AND IMPLEMENT IT.
IT SHOULD BE AS SEAMLESS AS
WI-FI AND THAT'S THE GOAL AND
IT'S NOW CALLED MATTER,
M-A-T-T-E-R AND THEY'RE WORKING
ON IT THIS MONTH IF YOU WANT TO
LOOK IT UP.
ANOTHER EXAMPLE IS I WORKED ON
ANOTHER GROUP IN TECHNOLOGY AND
ALS AND WHEN THE NEW TOBY
SOLUTION CAME OUT ON iPAD, WE
QUICKLY GOT IT IN THE HANDS OF
FOLKS WE THOUGHT COULD BENEFIT
FROM IT AND THERE WAS ONE GREAT
PERSON WHO WAS VERY INVOLVED IN
USING IT, LOVED THE SOLUTION BUT
REALLY HAD TROUBLE WITH THE
SCROLLI
SCROLLINGFY -- SCROLLING
FEATURE.
I WAS ABLE TO CONNECT THAT PAL
WITH THE ENGINEER WORKED ON
DEVELOPING THE PROJECT AND A GOT
AN E-MAIL THIS WEEK SAYING THEY
FIXED IT AND THE PAL WHO IT
IMPACTS COULDN'T BE HAPPIER.
THOSE ARE TWO EXAMPLES I WANTED
TO SHARE BUT I KNOW WE HAVE A
LONG WAY TO GO.
>>THE LARGE COMPANIES THAT
STEVE WORKS WITH, MOST OF THEM
HAVE PROJECTS RELATED TO
ASSISTIVE TECHNOLOGY.
FOR EXAMPLE, AND THERE'S A
PROJECT FOR WHEEL CHAIRS AT
MICROSOFT RESEARCH AND EUPHORIA
AND TRANSLATING SPEECH IS A
GOOGLE PROJECT.
THERE'S PROJECTS AVAILABLE FROM
MOST THE BIG COMPANIES AND
LOOKING FOR PALS PARTICIPATION.
>>CAN WE EXPECT TO SEE BRAIN
COMPUTER INTERFACE IN THE
MARKETPLACE FOR PEOPLE LIVING
WITH ALS?
THERE'S TWO TYPES OF BRAIN
INTERFACE.
ONE IS IMPLANTABLE WHERE THEY
PUT A CIRCUIT BOARD ON THE
SURFACE OF THE BRAIN AND GET
SIGNALS THAT WAY AND ANOTHER IS
NON-INVASIVE WHERE A PERSON
WEARS AN EEG CAP AND PICK UP
BRAIN SIGNAL THAT WAY.
BOTH SYSTEMS ARE TRYING TO GET
THE INTENT OF WHAT THE PERSON'S
SPEAKING OR CONTROLLING ROBOT OR
COMPUTER AND THE LIKE.
LIKE THE TRANSLATIONAL GROUP
SAID YESTERDAY THAT IT TAKES
TIME TO BRING SOMETHING TO
MARKET, THE SAME IS HAPPENING
WITH THE GROUPS THAT ARE TRYING
TO ACQUIRE BRAIN SIGNALS AND
INTERPRET THEM AND MAKE THEM
FUNCTION.
IT'S A COMPLICATED TASK.
THERE ARE SOME SIMPLE SYMPTOMS
AVAILABLE ON THE MARKET FOR
PEOPLE TO TRY THAT ARE THE
NON-INVASIVE SYMPTOMS.
YOU CAN TRY A NON-INVASIVE
COMPUTER SYSTEM AND WE HAVE
EVIDENCE THEY WORK IN A
COMMUNICATION DEVICE USING
BCI2000.
MOST THE WORK IS STILL IN THE
DEVELOPMENT STAGE AND WILL MOVE
TOWARDS IMPLEMENTATION IN THE
FUTURE.
>>HOW IS QUALITY OF LIFE FOR
PEOPLE LIVING WITH ALS AFFECTED
BY RACE AND GENDER?
>>THINK WE SEE ALS QUALITY OF
LIFE IMPACT RACE AND HOW PEOPLE
HAVE ACCESS TO CARE HOW PATIENTS
ARE BEING DIAGNOSED AND WHERE
PEOPLE ARE SEEN BY PROVIDERS.
I DID AN ETHNO GRAPHIC STUDY
WITH PATIENTS WIN ALS AND MOST
THE MOST WERE TOLD BY THEIR
PROVIDER AT SOME POINT THEY SAW
ALS AS A WHITE DISEASE AND THAT
BLACK PEOPLE COULD NOT GET IT.
SO THEY WERE MISSED DIAGNOSED
NOT BECAUSE OF A DOCTOR'S ERROR,
THEY WERE TRYING OATH THINGS AND
FOR SOME IT WAS COMING BACK TO
RACE AND DELETERIOUS AFFECTS AND
SOME WERE AFRAID TO GIVE THE
DIAGNOSIS BECAUSE IT WOULD
CHANGE THAT PERSON'S LIFE AND
BECAUSE OF THAT FEAR THEY'RE NOT
DIAGNOSING PATIENTS SHOWING
CLEAR UPPER AND LOWER MOTOR
NEURON SYMPTOMS AND WEEDED OUT
FROM OTHER DISEASES.
I ALSO THINK ONE OF THE
CHALLENGES THAT WE'RE SEEING
WITH RACE AND GENDER AS IT
PERTAINS TO QUALITY OF LIFE IS
THAT PEOPLE ARE NOT GETTING
ACCESS TO TREATMENT SOON ENOUGH
AND IF YOU'RE DIAGNOSED YOU
LIKELY WON'T GET DEVICE TO HELP
YOU BREATHE OR OTHER ASSISTIVE
TECHNOLOGIES EVEN THINKING OF
THE SPEECH TECHNOLOGIES WE
TALKED ABOUT IF YOU'RE DIAGNOSED
SO LATE YOU'RE NOT LONGER ABLE
TO SPEAK, SOME OF THE BANKING
SYSTEMS AREN'T GOING TO WORK FOR
YOU IN THE SAME WAY.
IF YOU DON'T HAVE ACCESS TO
RESOURCES BECAUSE YOU'RE AT THE
INTERSECTION OF LOW ECONOMIC
STATUS AND GENDER AND RACE, IT'S
GOING TO BE HARD TO ACCESS SOME
OF THE OTHER DEVICES THAT WILL
MAKE LIFE EASIER WITH THE
DISEASE.
SO I THINK IF WE ARE THINKING
ABOUT STEVE'S COMMENTS AT THE
ENDS OF OUR PRESENTATION, IF
YOU'RE THINKING ABOUT ALS
BECOMING A LIVABLE DISEASE THEN
WE HAVE TO MAKE SURE THAT WE'RE
TAPPING INTO ALL GROUPS THAT ARE
UNDER SERVED SO WE CAN ENSURE
THESE INDIVIDUALS ARE THEN ABLE
TO LIVE LONGER LIVES WITH THE
ILLNESS.
>>WHEN WE TALK ABOUT AUTOMATION
YOU'VE BEEN INVOLVED IN THAT AND
PERHAPS YOU'D LIKE TO COMMENT A
LITTLE BIT ON THAT.
>>I SERVE ON THE ADVISORY
COMMITTEE AND THEY'RE FOCUSSED
ON THIS AND STEVE GLEESON WANTS
VERY MUCH TO PARTICIPATE AND
THERE'S AN ARMY OF FOLKS THAT
THAT WANT TO MAKE SURE THERE'S A
GREAT QUALITY OF LIFE AND
CONSTANTLY LOOKING FOR SOLUTIONS
THAT HAVE MEANINGFUL CHANGE.
THEY DO GREAT WORK I HAVE TO BE
CAREFUL BECAUSE I'M UNDER N.D.A.
WITH SOME OF THE WORK WE'RE
DOING BUT I KNOW THEIR HARD WORK
WILL SHOW BENEFIT IN A LOT OF
AREAS THAT ARE INNOVATIVE AND
YET TO BE REALIZED AND SO STAY
TUNED AND AND I DON'T WANT TO
UNDERESTIMATE HOW NEW
TECHNOLOGIES ARE EXPENSIVE AND
WE HAVE TO PROVIDE FUNDING AND
ACCESS TO FOLKS AS THESE
EXCITING THINGS COME TO MARKET
BECAUSE IT WOULD BE A SHAME IF
THEY'RE THERE AND THE BARRIERS
OF GETTING TO USE THEM IS SIMPLY
COST.
>>ABSOLUTELY.
>>AND PEOPLE HAVE LOOKED AT THE
TRUE COST OF HAVING ALS AND
LIVING WITH IT AND THE TRUE COST
OF THE CLINICIAN WHO IS
RENDERING THE CARE.
AND 80% OF THE CLINICS LOSE
MONEY.
ONLY 20% OR ONLY 20% KNOW THEY
BREAK EVEN.
MORE THAN 90% OF US HAVE TO RELY
ON NON-PROFIT GROUPS WHO SUPPORT
US.
PHILANTHROPY.
AROUND THIS IS FOR PCORI OR
PUBLIC HEALTH TO LOOK AT THE
COST OF HAVING THE DISEASE.
THANK YOU, STEVE.
EVERYTHING'S PRETTY COSTLY.
WE'RE GOING MOVE ON WITH OUR
NEXT QUESTION I'LL DIRECT THIS
TO EMILY.
CAN WE RESEARCH THE ROLE OF
EXERCISE AND DIET TO MAINTAIN
AND IMPROVE QUALITY OF LIFE?
>>I'M HAPPY TO TAKE THE
QUESTION.
IT'S A PASSION PROJECT OF MINE.
AS I BRIEFLY HIGHLIGHTED WHEN WE
TALKED ABOUT SYMPTOM MANAGEMENT
APPROACHES FOR THE PRIORITY,
THIS HISTORICAL REACTIVE
MANAGEMENT APPROACH, THERE'S A
FEW ISSUES.
FIRST IT REALIZE ON THE CLINICAL
IDENTIFICATION OF A SYSTEM AS WE
ALL KNOW CAN BE VERY DELAYED IN
SOME CASES.
AND GIVEN THE LIKELIHOOD THERE
WILL BE BREATHING OR SWALLOWING
PROBLEMS THROUGHOUT THE DISEASE
PROGRESSION, SITTING BACK AND
WAITING TO APPLY MANAGEMENT
APPROACHES IN THE REACTIVE
MANNER IF THE COUGH DROPS BELOW
A MEASUREMENT AND LET'S START
USING COUGH ASSIST, ETCETERA AND
WAITING FOR THAT INTERVENTION
PUT PALS AND CALS IN THIS MORE
THAN NECESSARY VULNERABLE
SCENARIO WHERE THE DISEASE IS
DICTATE
DICTATING AND DOESN'T TAKE
ADVANTAGE OF BE AN EARLY WINDOW
OF OPPORTUNITY I'VE NOW SEEN
TIME AND TIME AGAIN TO INCREASE
THE FUNCTIONAL OR PHYSIO LOGIC
RESEARCH AND IMPROVE STRENGTH
FOR VITAL FUNCTION SUCH AS
EATING, DRINKING, BREATHING.
WE TYPICALLY USE THE ANALOGY TO
PALS AND CALS IN EXPLAINING OUR
STRATEGY AND WE'RE INCREASING
THEIR SAVINGS ACCOUNT IF YOU
WILL PRIOR TO THE INEVITABLE
DECLINES AND PHYSIOLOGIC
PROCESSES WE'RE REFERRING TO AND
GIVING THEM A HIGHER BASELINE OF
WHICH UNFORTUNATELY THERE WILL
BE SOME DECLINE FROM THAT AND
CLUMPING THE SYSTEM UP AND WE
PREFERRED TO HAND OVER THE
DECISION MAKING TO THE PATIENT
WHO WE FIND FEELS EMPOWERING AND
THE DEVIL'S IN THE DETAIL.
IT'S NOT A ONE-SIZE FITS ALL.
I HAVE TO SAY AND WE PUBLISHED
ABOUT SIX PAPERS IN THE AREA AND
OTHERS HAVE AS WELL AND THE USE
OF RESPIRATORY STRENGTH TRAINING
AND BREATH STACKING AND WE HAVE
WORK LOOKING AT THE COMBINATION
OF THE TWO THINGS TO IMPROVE
LUNG VOLUME AND AIRWAY CLEARANCE
CAPACITY PRIOR TO EMERGENCE OF
ANY ISSUES WE BELIEVE IS THE
BEST APPROACH AND HAVING BEEN A
PART OF THIS PANEL THE PAST TWO
DAYS I STARTED THINKING ABOUT IN
THE GENETIC CARRIERS CAN WE DO
MORE AND EXERCISING THESE FOLKS
BECAUSE WE KNOW THERE'LL BE A
DIFFERENT LINE SO WHY WOULD YOU
WAIT AND THE SIMILAR ANALOGY IS
HERE.
.
>>THIS IS FROM A QUESTION.
WE TALK ABOUT SYMPTOMS AND
FUNCTIONS AND THAT'S IMPORTANT
BUT IT SEEMS TO EXCLUDE EFFORTS
FOCUSSED ON WELLNESS, JOY AND
ENGAGEMENT FOR PEOPLE LIVING
WITH ALS AT ANY LEVEL OF
FUNCTION AND SEEMS TO EXCLUDE
RESEARCH FROM INFECTIONS FROM
ALS AND HOW CAN WE MAKE SURE
THOSE ELEMENTS ARE INCLUDED TOO?
>>WE TALKED ABOUT SYMPTOM
FUNCTION AND MENTAL HEALTH AND
PSYCHOLOGICAL ASPECTS EVEN ONCE
THE PANDEMIC HIT IT'S BEEN
DIFFICULT FOR EVERYONE INVOLVED
IN ALS AND PEOPLE PROVIDING CARE
AND PEOPLE LIVING WITH THE
DISEASE.
ONE THE CHALLENGES IS THAT MANY
OF THE QUALITY OF LIFE
INSTRUMENTS THAT HAVE BEEN USED
ARE SOFTEN USED IN CLINICAL
TRIALS AND TRACK MORE WITH
HEALTH SPECIFIC ITEMS.
AND WELL BEING IS AN IMPORTANT
ASPECT.
THERE IS SOME IMPORTANCE IN
UNDERSTANDING WELL BEING IN THE
CONTEXT OF QUALITY OF LIFE AND
THAT IS THE EMBEDDED IN OUR
WORKING GROUP.
WELL BEING IS SUCH A BROAD TERM.
WE TALK ABOUT PHYSICAL WELL
BEING AND THERE'S ASPECTS OF
ECONOMIC AND SOCIAL WELL BEING
AND LIFE SATISFACTION AND WELL
BEING ASSOCIATED WITH ENGAGEMENT
OF ACTIVITIES AND THERE ARE
SCALES THROUGH NHANES AND NIEHS
THAT LOOK AT WELL BEING AND THIS
IS WITH THE INTENT OF LOOKING AT
THE SPECIFIC GOAL AND THERE'S
IMPLEMENTED WITH WHATEVER GOAL
YOU'RE TRYING TO LOOK FOR.
I THINK IS AN IMPORTANT ASPECT
THAT CAN BE EVALUATED MORE THAN
JUST THE PHYSICAL WHICH A LOT OF
OUR EFFORTS ESPECIALLY IN
RESEARCH HAVE BEEN FOCUSSED ON.
THE SECOND POINT AND THIS TAILS
IN WITH THE HEALTH ECONOMIC
IMPACT OF LIVING WITH ALS.
IT MIGHT BE THAT WE INCORPORATED
THE QUALITY OF LIFE SCOPE OR
THIS COULD BE BETTER SUITED IN
MAKING IMPROVEMENTS IN RESEARCH
AND OFTEN IN CLINICAL TRIALS
THERE'S COLLECTION OF
HOSPITALIZATION AND FALLS AND IT
RELATES TO AN INTERVENTION OR
EXPERIMENTAL THERAPY AND THAT'S
TO UNDERSTAND HOW WE CAN DERIVE
NOT ONLY THE EFFICACY AND SAFETY
AND TOLERABILITY OF THAT
EDUCAT
INTERVENTION AND THE HEALTH
ECONOMIC IMPACT.
BEYOND LOOKING AT IT IN
EXPERIMENTAL CLINICAL TRIALS IT
OPENS ANOTHER QUESTION BECAUSE A
LOT OF WHAT THE STRATEGIC
PLANNING HAS FOCUSSED ON IS
RESEARCH AND RESEARCH-BASED
QUESTIONS BUT CLINICAL CARE,
FALLS AND INFECTIONS AS IT
PERTAINS TO CLINIC CARE IS
ANOTHER AREA WHERE I THINK WE
CAN CERTAINLY INVESTIGATE AND I
KNOW THAT THE ALS ASSOCIATION
HAS HAD AN INTEREST AND
INITIATIVE TO LOOK AT QUALITY
IMPROVEMENTS OF CARE BUT THIS IS
ALSO ANOTHER IMPORTANT TOPIC.
>>THE NEXT PERSON, JEAN AND
STEVE I THINK MAY WANT TO WEIGH
IN.
DO CAREGIVERS AND PEOPLE LIVING
WITH ALS FIND IT BENEFICIAL TO
PARTICIPATE IN ONLINE PATIENT
ADVOCACY GROUPS LIKE PATIENTS
LIKE ME?
AND WHAT ROLE SHOULD IT PLAY AND
LET US KNOW YOUR FEELINGS AND
TAKE INTO ACCOUNT PALS AND CALS.
>>THIS IS AN INTERESTING POINT
AN THERE'S ANOTHER QUESTION WE
MAY NOT GET TO THAT ALSO TOUCHED
ON THIS AND IT'S THE CONTEXT OF
PEOPLE SHARING THEIR TIPS AND
TRICKS CARING FOR THEIR LOVED
ONE WITH ALS.
WE MENTIONED EVIDENCE-BASED
INFORMATION AND RESOURCES.
AND ONLINE COMMUNITY AND SOCIAL
MEDIA IS A RESOURCE THAT CAN BE
TRANSFORMATIVE FOR PEOPLE LIVING
WITH ALS AND CAREGIVERS AND
PEOPLE AT RISK AT GENETIC ALS.
I THINK IT'S IMPORTANT FOR ANY
LISTING OF RESOURCES TO INCLUDE
THAT.
I KNOW WE SHOULD BE CAREFUL WHEN
THE GOVERNMENT OR CLINICAL
EXPERTS ARE GIVING INFORMATION
THAT IS EVIDENCE-BASED AND
PEOPLE WILL RELY ON IT AT
FULL-BASED VALUE AND NEED TO
ACCEPT THAT IT'S FULLY PROVEN.
CERTAINLY IN SOCIAL MEDIA OR
OTHER THINGS THAT'S NOT
NECESSARILY HAPPENING.
AND THIS IS JUST LIKE TALKING TO
OTHER PEOPLE AND YOU'LL HEAR
INTERESTING MENTION AND TALKING
TO OTHERS IS BENEFICIAL.
AND ENSURING THESE THINGS EXIST
AND PEOPLE WANT TO PARTICIPATE
IS A GOOD THING.
>>I AGREE WITH EVERYTHING YOU
SAID.
THIS IS PROBABLY HIGH ON THE
LIST OF THINGS FOLKS ASK ME
ABOUT WHEN THEY FIND OUT WE'RE
GOING TO DO AND LEARN A LITTLE
BIT.
THEY WANT TO KNOW RESOURCES AND
COMMUNITY GROUPS AND SUPPORT
GROUPS THEY CAN BELONG TO AND
AGAIN THIS FALLS UNDER THE
CATEGORY OF PARTICIPATION.
NOT ONLY DO WE WANT THEM TO
PARTICIPATE WHEN THEY'RE NEWLY
DIAGNOSED BUT STAY ENGAGED ALONG
THE WAY BECAUSE THAT CERTAINLY
HAS IMPACT ON THE QUALITY OF
LIFE AND FEELING THAT THEY'RE
CONNECTING WITH OTHERS OR
SHARING THEIR LIVED EXPERIENCE
AND WE CAN LEAN ON EACH OTHER
AND HELP EACH OTHER THROUGH
DIFFICULT JOURNEYS.
>>I ALSO WANTED TO POP IN AND
SAY ANOTHER REALLY GREAT BENEFIT
OF SPACES IN PATIENTS LIKE ME IS
FOR THE UNDER REPRESENTS
POPULATIONS.
THEY'RE USUALLY ABLE TO FIND
THESE SPACES AND FIND COMMUNITY
SO FOR YOUNGER PEOPLE WITH ALS,
BLACK PEOPLE WITH ALS,
INDIGENOUS FOLK WITH ALS, THEY
FOUND SOME OF THESE SPACES SO I
THINK IN ANSWERING THE SECOND
PART OF THE QUESTION OF HOW CAN
THE SPACES BE IMPROVED, I THINK
SOME OF THESE NON-PROFITS AND
COMMUNITY ORGANIZATIONS THAT CAN
MAYBE TAKE UP SOME OF THESE
SPACES AND MAKING THEM MORE
INTENTIONAL COMMUNITY SPACES
THAT MAYBE AREN'T SUPPORT GROUPS
BY NAME BUT ARE SUPPORT SPACES
WHERE PEOPLE WITH ALS COULD BE
WITH ONE ANOTHER COULD BE VERY
USEFUL.
>>AND DR. CARTER, WHILE ON THAT
THOUGHT A QUESTION CAME IN AND I
THINK THE LAST ONE WE'LL HAVE
TIME FOR, SOMEBODY FROM INDUSTRY
ASKED -- I'M LOSING IT BUT HOW
DO WE ENSURE PEOPLE ARE ABLE TO
HAVE BETTER REPRESENTATION IN
CLINICAL TRIALS?
>>IT'S A GREAT QUESTION.
I THINK I WOULD ARGUE THAT I
THINK THE P.I.s JUST HAVE TO
MAKE IT A PART OF THEIR PLAN.
I WAS RECENTLY ON OTHER OF
ANOTHER ORGANIZATION PANEL WITH
A RESEARCHER FROM EMORY
UNIVERSITY AND THEY HAVE NOW
HIRED SOMEONE EXPLICITLY TO
SUPPORT THE INITIATIVES THEY
WANT TO DO.
THIS PERSON ALREADY HAS
RELATIONSHIP WITH THE COMMUNITY
AND ENGAGED WITH THE COMMUNITY
AND BUILT TRUST WITH THE
COMMUNITY.
SHE'S ABLE TO ANCHOR THOSE
RELATIONSHIPS AND BUILD THE
TRUST TO BE ABLE TO GET MORE
COMMUNITIES OF COLOR INTO THOSE.
THINK IT'S INCUMBENT ON ANYONE
WANTING INDUSTRY OR P.I.s AT
CLINIC RESEARCH LABS, IF THIS IS
A PRIORITY YOU HAVE TO WANT TO
PAY FOR THESE THINGS AND REALLY
SUPPORT THE MOVEMENT OF FUNDS TO
SUPPORT NOT ONLY A PERSON THAT'S
ACTUALLY HIRED FOR THIS JOB BUT
KNOWING THE USUAL METHODS WE
UTILIZE FOR RESEARCH DEVELOPMENT
AREN'T GOING TO BE THE SAME FOR
UNDER REPRESENTED POPULATIONS.
ARE YOU GOING TO COMMUNITY
EVENTS?
ARE GOING TO CHURCHES AND
CALLING PEOPLE AND SENDING TEXT
MESSAGES?
ARE WE LOOKING OUTSIDE THE BOX
AND I HAVE A LONGER ANSWER BUT I
WANT TO BE RESPECTFUL OF TIME.
THANK YOU FOR THE QUESTION.
>>THANK YOU SO MUCH.
>>AND REACHING OUT TO THE
COMMUNITY TO GET UNDER
REPRESENTED MINORITIES NOT ONLY
AFRICAN AMERICAN BUT ALSO
HISPANIC AND OTHERS.
LUCKILY, I CAN LEVERAGE SOME OF
THE MECHANISMS ALREADY AVAILABLE
FOR THAT BECAUSE WE'RE COMMITTED
TO THAT.
I THINK THAT'S ALL WE HAVE TIME
FOR IN THE SESSION.
I WANT TO THANK MY COLLEAGUES IN
THE WORKING GROUP.
YOU GUYS HAVE BEEN SO TERRIFIC
TO WORK WITH AND YOUR COMMITMENT
TO THIS AREA AND TO ALS, PERIOD
IS JUST UNBELIEVABLE.
IT'S BEEN A PRIVILEGE.
THANK YOU.
FOR EVERYBODY OUT THERE, WE'LL
CONTINUE COLLECTIVE QUESTIONS
THROUGH THE ENGAGEMENT FORM ON
THE WEBSITE AND WE'RE LOOKING
FORWARD TO CONTINUING TO SHAPE
THE STRATEGIC PLAN AND SOME
PEOPLE BROUGHT UP PRIORITIZING
THE PRIORITIES AS WELL AS HOW DO
WE GET THE MONEY FOR ALL THIS.
WE CAN HAVE A WHOLE CONVERSATION
ABOUT LEVERAGING TECHNOLOGY AND
WHERE'S THE MONEY GOING TO COME
FROM.
THE TECHNOLOGY'S THERE WE JUST
NEED THE MONEY TO APPLY IT AND
GET THE RIGHT ENGINEERS WORKING.
THANK YOU, EVERYONE.
WE'LL MOVE FORWARD WITH THE NEXT
SECTION.
>>WELCOME BACK.
EVERYBODY.
THIS WAS A FANTASTIC SESSION
THIS MORNING AND CONGRATULATIONS
TO OUR WORKING GROUP AND ITS WAS
INSPIRING AND FANTASTIC GREAT
DISCUSSION AND WONDERFUL
QUESTIONS.
SO WITH THAT IN MIND, WE'RE
GOING TO MOVE ON TO OUR LAST
WORKING GROUP SESSION FOR THIS
WORKSHOP WHICH IS CO-CHAIRED BY
LISA GOLD AND DR. DOCTOROFF TO
UNDERSTAND COLLABORATIONS AN
PARTNERSHIPS AND FIND
OPPORTUNITIES TO WORK TOGETHER
MORE EFFICIENTLY AND WITH THAT
HAND IT OVER TO DAN AND LISA.
>>THANK YOU, RITA.
ANOTHER REMINDER YOU CAN ENTER
QUESTIONS IN THE ZOOM Q&A WORK
OR QUESTIONS AND COMMENTS.
I WANT TO THANK YOU.
MY NAME IS LISA GOLD I'LL
INTRODUCE MYSELF AND THEN ASK
DAN TO INTRODUCE HIMSELF.
I'M A NEURO SCIENTIST BY
TRAINING.
I STARTED IN ACADEMIC RESEARCH
AND SPENT 21 YEARS IN THE
PHARMACEUTICAL INDUSTRY.
MY MOTHER DIED OF ALS IN 19995
AND SO LIKE MANY PEOPLE ON THE
CALL THIS IS PERSONAL TO ME.
AND NOW I'LL TURN IT OVER TO
DAN.
>>THANK YOU, SO MUCH, LISA.
THANKS TO OUR WONDERFUL TEAM
MEMBERS.
ALL OF WHOLE YOU'LL HAVE THE
CHANCE TO MEET IN A LITTLE BIT.
I'M DAN DOCTOROFF.
MY FATHER DIED OF ALS IN 2002.
MY UNCLE, HIS BROTHER, DIED IN
2011.
AND WHEN MY UNCLE DIED, I
THOUGHT I HAD TO REALLY DO
SOMETHING.
I ASSUMED WHEN MY FATHER DIED IT
WAS JUST A FLUKE.
AT IT TURNS OUT MY UNCLE AND
FATHER AND UNCLE ALL HAD THE C9
MUTATION.
I'VE BEEN THE ETERNAL OPTIMIST,
ASSUMED I'D NEVER GET IT BUT
WHEN MY UNCLE DIED I STARTED
TARGET ALS.
THE WHOLE MISSION IS DESIGNED TO
ACCELERATE RESEARCH IN THE
SUCCESSFUL CLINICAL TRIALS AND
WE GO ABOUT IT MULTIPLE WAYS
INCLUDING FUNDING CONSORTIUM
RESEARCHERS OFTEN LED BY
INDUSTRY ON IMPORTANT VACCINE
PROBLEMS IN ALS RESEARCH.
WE ALSO FUND TOOLS AND RESOURCES
THAT ANYONE IN THE WORLD CAN
DRAW ON.
THINGS LIKE STEM CELL LINES AND
MOUSE MODELS AND POSTMORTEM
TISSUE SAMPLES AND BIO FLUIDS
AMONG OTHERS.
WE'RE VERY FOCUSSED ON GETTING
DERIVATIVE INVOLVED AND WE'RE
ACTIVELY ENGAGED WITH 123
COMPANIES OR VENTURE CAPITAL
FIRMS WHICH SHOULD FOR EVERYBODY
BE AN INCREDIBLY POSITIVE SIGN
THAT WE AS A FIELD ARE MAKING
SIGNIFICANT PROGRESS.
WE ALSO HAVE PARTNERSHIPS WITH
OTHER DISEASES WE HAVE VENTURES
WITH THE GATES AND ALZHEIMER'S
DISCOVERY FUND WORKING ON THINGS
LIKE BIOMARKER AND ANIMAL MODELS
AND OTHER THINGS.
AS I SAID, I NEVER EXPECTED I'D
GET ALS.
ABOUT A YEAR AGO I WAS
DIAGNOSED.
INTERESTINGLY, I DO NOT HAVE THE
C9 MUTATION SO I EITHER HAVE
ANOTHER GENETIC FORM THAT HASN'T
BEEN DISCOVERED YET OR SPORADIC
ALS.
BUT WHEN I WAS DIAGNOSED I
DECIDED TO RETIRE FROM THE
COMPANY I FORMED WITH GOOGLE AND
STEP BACK FROM MOST OF MY OTHER
INVOLVEMENT AND FOCUS ON SCALING
UP TARGET ALS AND WORKING WITH
THE WHOLE COMMUNITY TO ACC
ACCELERATE TREATMENT RESEARCH
INTO SUCCESSFUL TREATMENTS.
THANK YOU VERY MUCH AND THIS HAS
BEEN A GREAT HONOR TO BE A PART
OF THIS.
>>THANKS, DAN.
YOU SEE ON THE SLIDE THE MEMBERS
OF OUR COMMITTEE AND WE'LL ASK
THEM TO INTRODUCE THEMSELVES AT
THE END OF THE PRESENTATION TO
KICK OFF OUR DISCUSSION.
I WANT TO NOTE ALLISON BULAT AND
JOHN RAVITS ARE NOT ABLE TO JOIN
US AND WHILE DAN AND I WILL BE
GIVING THE PRESENTATION, THESE
ARE THE PEOPLE WHO PROPOSED THE
IDEAS AND DEVELOPED THE IDEAS
AND DISCUSSED THE IDEAS AND THEN
SELECTED THE TOP THREE
PRIORITIES WE'LL BE PRESENTING
TODAY.
OUR COMMITTEE IS MADE UP OF
BROAD REPRESENTATION OF THE ALS
COMMUNITY.
THAT INCLUDES INDIVIDUALS FROM
INDUSTRY, CLINICIANS, CLINICAL
AND PRE-CLINICAL RESEARCHERS AND
PEOPLE LIVING WITH ALS OR
MEMBERS WITH FAMILY MEMBERS
LIVING WITH ALS AND
REPRESENTATIVES OF THE FDA AND
NIH AND THESE ARE THE KEY GROUPS
TO BE INVOLVED IN ANY
COLLABORATION AND PARTNERSHIP.
THERE'S BEEN PROGRESS IN
ACCELERATING ALS RESEARCH INTO
CLINICAL TRIALS AND WE'RE
LOOKING TO TAKE ADVANTAGE AND
CONTINUE TO IMPROVE ON THE
PROCESS GOING FORWARD.
PROGRESS HAS OCCURRED IN
BRINGING TECHNOLOGIES IN THE
FIELD, NEW DISCOVERIES OF
GENETIC AND BIOLOGICAL PATHWAYS
TA CAUSE MOTOR NEURONS TO DIE
AND THE ENGAGEMENT OF THE BIO
TECH AND PHARMACEUTICAL
INDUSTRY.
FROM THE REQUEST OF INFORMATION,
INPUT FROM THE ALS COMMUNITY
HIGHLIGHTED HIGH AREAS OF
PRIORITY FOR COLLABORATIONS AND
PARTNERSHIPS THAT INCLUDE
URGENCY, ACCESS AND COORDINATION
OF RESEARCH EFFORTS.
WITH THIS IN MIND WE SELT A GOAL
TO LOOK AT SYNERGIES ACROSS
GOVERNMENTAL AND
NON-GOVERNMENTAL ORGANIZATIONS
AND TRANSLATE THE IDEA THE LAB
TO CLINIC AND CARE MODELS.
DAN IS GOING TO INTRODUCE YOU TO
OUR TOP THREE PRIORITIES.
THERE'S A LOT IN THIS STATEMENT
HERE THAT IS IMPORTANT TO
EMPHASIZE.
FIRST IS IMPORTANT POTENTIAL
SYNERGIES ACROSS ORGANIZATIONS
AND PARTNERSHIP AND WANT TO
LEVERAGE SHARED RESOURCES ALL
WITH THE GOAL OF ACCELERATING
THE TRANSLATION OF IDEA FROM THE
LAB TO CLINIC AND CLINICAL AND
CARE MODELS.
SOMETIMES PEOPLE DONE WORK
TOGETHER AND SHARE RESOURCES
WITH THE GOAL OF ACCELERATING
RESEARCH IN TO SUCCESSFUL
CLINICAL TRIALS AND CARE MODELS.
I THINK IT FRAMES THE DISCUSSION
DISCUSSION WE'RE ABOUT TO HAVE.
THEN WE'LL TALK MORE DETAIL
ABOUT THEM AND WE WILL ENGAGE
QUESTIONS AND ANSWERS.
FIRST IS TO ESTABLISH A
MULTI-MODEL DATA PLATFORM WE'RE
INCLUDING PATIENT DATA AND
CLINICAL DATA GENOMES, IMAGING,
BIO FLUIDS, SPECIMENS, BLOOD
REPOSITORY WITH A FOCUS ON
DEFINING THE STANDARDS OF FUTURE
DATA COLLECTION THAT WILL
PROMOTE OPEN DATA ANDS A.I. AND
MACHINE EARNING AND LOOK AT THE
HEAT HETEROGENOUS DISEASE AND
DEVELOP A PLATFORM OF RESOURCS
TO MAKE WORKING ON THE DISEASE
LOWER THE BARRIERS TO ENTRY.
WE'RE TALKINGS ANTIBODIES AND
VIRAL VECTORS TO IS BEING SELL
RATE RESEARCH AND LOOK AT WHAT
IS PRIORITIZED FOR FUTURE
SCIENTIFIC EFFORTS.
THE THIRD PRIORITY A FRAMEWORK
FOR RESEARCH COLLABORATION
ACROSS ACADEME ANDNON-PROFIT
ORGANIZAT
ORGANIZATIONS I'M GOING TO GO
INTO MORE KE TAIL ON EACH OF THE
FIRST TWO THEN LISA WILL TALK
ABOUT PRIORITY THREE.
WE'RE TALKING ABOUT A
MULTI-MOD
MULTI-MODELAL AND QUE NEED TO
BETTER UNDERSTAND THE DISEASE.
AND OUR HYPOTHESIS AND TOOLS FOR
GREATER EXTRACTION TECHNOLOGIES
AND A.I. AND MACHINE LEARNING WE
KCAN LOCK AT DEVELOPMENT AND WE
NEED TO HAVE COMPREHENSIVE
SOURCES OF DATA FROM LARGE AND
DIVERSE COHORTS OF PATIENTS.
THIS IS EXPENSIVE AND
COMPLICATED BUT WHAT WILL MAKE
THIS CRITICAL AND WHAT IS
NECESSARY TO ACHIEVE THIS AROUND
THE NEXT STEPS.
FIRST WE HAVE TO AGREE ON WHAT
THE ELEMENTS ARE FOR THE
MULTI-MODAL DATABASE AND THAT
INVOLVES BRINGING THE COMMUNITY
TOGET
TOGETHERAGE WILL NEED CONSENT ON
DATA COLLECTED RESPONSIVELY AND
NEED STANDARDS FOR ALL DATA
TYPES INCLUDING EMERGING DATA
TYPES AND GOING TO NEED A
CONSISTENT SET OF PROTOCOLS AND
LOOK AT THE POPULATION IN
RESEARCH DATABASES.
AS WE HEARD YESTERDAY THERE'S A
WHOLE SERIES OF DATABASES THAT
ARE IN THE PROCESS OF BEING
COLLEC
COLLECTED THERE'S ONES AROUND
THE WORLD BUT I THINK WE AGREE
THERE ARE GAPS.
AND THEY ARE NOT CONSISTENT AND
THE COLLECTION PROCESSES ARE NOT
THE SAME.
SO WHAT WE NEED TO DO IS LOOK AT
WHAT THE DATABASE IS AND HOW WE
COLLECT THE DATA AND THEN
COLLECT IT AND TIE WHATEVER
DATABASES THAT AND LOOK AT THE
DATA ELEMENTS TO BE CAPTURED.
AND WHAT IS OUT THERE AND HOW IT
CAN BE BROUGHT TOGETHER IN A
CONSISTENT STRATEGY.
WE HAVE TO CREATE THE MECHANISMS
FOR DATA SHARING.
AND SUBMIT TO THE SHARED DATA
PLATFORM AND PROVIDE CARROTS AND
STICKS TO MAKE SURE WE CAN
COLLECT IN A CONSISTENT MANNER
TO PROVIDING GUIDELINE AND
RESOURCE TO COLLECTION AND
CREATION OF DATA SETS ACROSS
STUDIES.
WE HAD MULTIPLE OPTIONS AND THIS
WAS BY FAR THE MOST AND THIS IS
IMPORTANT AND THIS IS NOT AN
INEXPENSIVE EFFORT AND HOPEFULLY
WE CAN DEVELOP A STRATEGY TO
FIND A WAY TO FUND THIS.
THE NEXT SLIDE IS TO DEVELOP
RESEARCH TOOLS AND PROTOCOLS AND
RESOURCES.
THE CRITICAL FACTOR WE'VE MADE
PROGRESS IN THE LAST DECADE IS
MORE PEOPLE FROM DIFFERENT FIELD
HAVE STARTED TO WORK ON ALS AND
THE EASE OF DOING RESEARCH HAS
GROWN GREATER.
WE NEED TO LOWER THE BARRIERS TO
ENTRY FOR RESEARCHERS BY
WIDENING THE FIELD.
THERE ARE TOOLS AND THERE'S LACK
OF AWARENESS WITH THE TOOLS AND
RESOUR
RESOURCES AND LACK OF PROTOCOLS
AND METHODOLOGIES TO CREATE AND
COLLECT AND USE THE TOOLS AND
RESOURCES.
WHAT WE BELIEVE IS NECESSARY IS
TO CENTRALIZE INFORMATION ON
TOOLS AND RESOURCES AND THAT WE
THINK WILL DRAW MORE PEOPLE IN
THE FIELD AND DO MAKE DOING
RESEARCH EASIER FOR EVERYONE WHO
IS ENGAGED.
WHAT WE SUGGEST IS TO FORM A
COMMITTEE THAT INCLUDES
REPRESENTATION FROM ACADEMIA AND
NONPROFITS AND GOVERNMENT THAT
WE HAVE TO LOOK AT THE TOOLS
NECESSARY TODAY AND POTENTIALLY
IN THE FUTURE IN ORDER TO DO
EFFECTIVE RESEARCH AND THAT CAN
INCLUDE THINGS LIKE BIO SAMPLES
AND ANIMAL MODELS ANTIBODIES,
STEM CELLS, ETCETERA.
WE NEED PROTOCOLS TO USE
CONSISTENT TOOLS AND RESOURCES
AND IDENTIFY GAPS IN TOOLS AND
RESOUR
RESOURCES AND USE THE TOOLS AND
MAKE SURE PEOPLE ARE USING THE
HIGHEST QUALITY TOOLS.
WE SEE THIS AS A PLATFORM BUT AS
AN INTERCONNECTED PLATFORM WHERE
THERE'LL BE MULTIPLE SOURCES OF
TOOLS AND HAVE CONSISTENT
POLICIES AND PROTOCOLS.
ONE THING THAT IS IMPORTANT AND
WE'VE SEEN THIS AT TARGET ALS,
IS IP.
IT WILL SLOW THINGS DOWN
DRAMATICALLY IF THESE PLATFORMS
AND THE CENTRALIZED PLATFORM
RETAIN RIGHTS TO DATA AND
INTERNATIONAL PROPERTY.
THESE NEED TO BE OPEN PLATFORMS
WHERE NO IP IS ACTUALLY TAKEN.
AGAIN, WE CAN USE THE NIH AND
IT'S FUNDING TO ENSURE FO AT
LEAST FOR PEOPLE FUNDED BY MIN
TO ENSURE WE UPLOAD HISTORICAL
INFORMATION AND WE'RE ALSO GOING
TO NEED AND TO SHARE INFORMATION
TO THE PLATFORM AS WELL.
AGAIN THE KEY THING IS LOWERING
THE BARRIERS TO ENTRY FOR
RESEARCHERS TO DO WHAT THEY DO
BEST.
THAT GOAL IS TO ACCELERATE
RESEARCH IN SUCCESSFUL CLINICAL
TRIALS.
LISA.
>>THANK YOU, DAN.
OUR THIRD PRIORITY WAS TO
ESTABLISH A FRAMEWORK FOR ALS
RESEARCH COLLABORATION.
AND AGAIN, WE'VE HAD A LOT OF
DISCUSSION ABOUT THIS ALREADY
HOW THERE IS A NEED TO BETTER
COORDINATE HOW THE ACADEMIC,
BIOPHARMACEUTICAL AND GOVERNMENT
SECTORS THAT PARTICIPATE IN ALS
RESEARCH AND THERAPY DEVELOPMENT
GENERATE, SHARE AND USE
KNOWLEDGE TO ADVANCE CRITICALLY
NEEDED THERAPIES.
SO THERE'S A LOT OF SEGMENTS OF
THE RESEARCH COMMUNITY THAT ARE
INVOLVED IN EFFORTS AND THERE'S
AN OPPORTUNITY FOR BETTER
COORDINATION SO THAT WE'RE ABLE
TO MOVE FORWARD MORE QUICKLY
WITH URGENCY THE COMMUNITY
DEMANDS AND REQUIRED.
THE HYPOTHESIS WE CAME UP WITH
WAS INCENTIVIZING ALS RESEARCH
STAKEHOLDERS FROM DISCOVERY OF
POSSIBLE THERAPEUTIC IDEAS TO
VALIDATION OF TARGETS AND
THROUGH DEVELOPMENT WILL HELP TO
FOCUS THE EFFORTS AND THEN
ACCELERATE THE TRANSLATION OF
IDEAS FROM THE LAB TO THE CLINIC
TO THE PEOPLE WITH ALS LIVING
WITH ALS TODAY.
AND THERE'S TWO EXAMPLES HERE OF
THESE TYPES OF RESEARCH
FRAMEWORKS OR COLLABORATIONS AND
YOU'VE HEARD ABOUT BOTH OF THEM
YESTERDAY.
THE CRITICAL PABLG FOR RARE
NEURODEGENERATIVE DISEASES
ANNOUNCED IN SEPTEMBER AND THE
ACCELERATING MEDICINES
PARTNERSHIP PROGRAM AND THOSE
ARE JUST TWO EXAMPLES AND ON THE
NEXT SLIDE I'LL PROVIDE A LITTLE
BIT MORE INFORMATION ABOUT ONE
OF THESE PARTNERSHIPS THAT HAS
BEEN VERY SUCCESSFUL RUN THREW
THE NIH PROGRAM AND ACCELERATED
MEDICINES, RAMP, LAUNCHED AS A
PUBLIC-PRIVATE PARTNERSHIP AMONG
NON PROFITS AND PHARMACEUTICAL
COMPANIES AND OTHER
ORGANIZATIONS TO TRANSFORM THE
MODEL FOR DEVELOPING NEW
DIAGNOSTICS AND TREATMENTS.
THERE'S A VARIETY OF PROGRAMS
THAT HAVE THESE ACROSS A RANGE
OF DISEASES AND MOST RELEVANT
FOR THIS GROUP IS ALZHEIMER'S
DISEASE AMP PROGRAM AND THEY'RE
ON A 2.0 PROGRAM FOR THAT.
THERE'S ONE ON PARKINSON'S
DISEASE AND SCHIZOPHRENIA.
THE AMP PARTNERS HAVE AN
INCREASING GOAL OF INCREASING
THE NUMBER OF NEW DIAGNOSTICS
AND THERAPIES FOR PATIENTS AND
TO REDUCE THE TIME AND COST OF
DEVELOPING THEM.
IT AIMS TO LOOK AT THE PATHWAYS
AND VALIDATE INFORMATION FOR THE
DEVELOPMENT OF MULTIPLE
THERAPIES.
THIS ENABLES THE BEST INFORMED
SCIENCE ACROSS ALL PARTICIPANTS.
AND THIS CROSS-SECTOR
PARTNERSHIPS ARE MANAGED
TYPICALLY THROUGH THE FOUNDATION
FOR THE NIH AND THE AMP PARTNERS
SHARE EXPERTISE AND RESOURCES IN
AN INTEGRATED GOVERNANCE
STRUCTURE.
WE HEARD A LOT AS WELL ABOUT NOT
WANTING TO REINVENT THE WHEEL
AND WE COULD UPDATE THE WHEEL
AND DEFINITELY NEED TO MAKE SURE
IT'S FIT FOR PURPOSE FOR ALS
RESEARCH.
FOR PRIORITY 3, THE KEYS TO
SUCCESS ARE BASICALLY
FACILITATING BETTER COORDINATION
AND ESPECIALLY BUILDING ON
SUCCESSFUL ONGOING PARTNERSHIPS.
AS MENTIONED BY DAN AND OTHERS,
THERE'S GREAT AND VERY PROMISING
AND SUCCESSFUL PARTNERSHIPS AND
COLLABORATION ARE ALREADY
ONGOING.
WHAT WE WANT TO DO IS FACILITATE
BETTER COORDINATION TO REALLY
LEVERAGE THE ONGOING SUCCESS OF
THOSE AND THE RESULTS RESEARCH.
AND INTEGRATE EXISTING
COLLABORATIONS SUCH AS TARGET
ALS OR THE ANSWER ALS AND
EVERYTHING ALS PROGRAMS AND
YOU'LL HEAR ABOUT OTHERS AND
LEVERAGING THE EFFORTS AND
OUTPUTS OF THOSE EXISTING
COLLABORATIONS.
AND THE FIRST STEP WOULD BE THEN
TO REVIEW THE PROGRAMS AND
INDEPENDENT ALS COLLABORATIONS
AND REALLY TRY TO IDENTIFY
LEARNINGS FROM THOSE THAT WILL
IMPROVE THE OVER ALL IMPACT AND
SPEED AS WELL AS TO IDENTIFY THE
SPECIFIC RESEARCH QUESTIONS THAT
ARE BEST ADDRESSED BY SUCH
FRAMEWORKS.
AND EARLY FOCUS AREAS WOULD
INCLUDE EVALUATION OF REGULATORY
ISSUES REGARDING SHARED PATIENT
DATA AND BIOLOGICS ACROSS
INDUSTRY AND I SAW A QUESTION
ABOUT SHARING PATIENT DATA
THAT'S A COMPLEX ISSUE.
CONSIDERATION OF MECHANISMS TO
ENCOURAGE AND SUPPORT ADDITIONAL
DATA SHARING BY SMALLER SOURCES
AS WELL AS PATIENTS WHILE
MAINTAINING DATA STANDARDS.
LEVERAGING NIH-FUNDED BIO SAMPLE
TO ADD NOVEL DATA AND SAMPLES
AND INVOLVING BIG DATA PARTNERS
AS DAN MENTIONED WITH A.I. AND
MUCH LEARNING TO DEVELOP NEEDED
TOOLS AND ALGORITHMS.
THESE ARE THE THINGS WE THINK
WOULD BE IMPORTANT NEXT STEPS
OBVIOUSLY ONCE THE FRAMEWORK IS
THERE THERE'LL BE INPUT FROM THE
PARTICIPANTS AND IDENTIFICATION
AND PRIORITIZATION OF THE KEY
FIRST NEXT STEPS.
THE NEXT SLIDE IS A SUMMARY.
OUR WORKING GROUP GOAL WAS TO
IDENTIFY THE MOST SYNERGIES
ACROSS GOVERNMENTAL AND NON OF
THIS GOVERNMENTAL ORGANIZATIONS
AND SHARE THE RESOURCES TO
TRANSLATE THE IDEAS FROM THE LAB
TO THE CLINIC AND CARE MODELS TO
THE ALS PATIENTS AND THEIR
FAMILIES.
WE EMPHASIZED WHAT WAS TAKEN
REGARDING URGENCY, ACCESS AND
COORDINATION OF RESEARCH
EFFORTS.
THE THREE HIGH PRIORITIES WERE
TO ESTABLISH A MULTI-MODAL DATA
PLATFORM AND DEVELOP A PLATFORM
FOR REPOSITORY OF RESEARCH
TOOLS, PROTOCOLS AND RESOURCES.
AND TO ESTABLISH A FRAMEWORK
FOR ALS RESEARCH COLLABORATION.
WE WANTED TO END WITH A
STATEMENT FROM THE COMMUNITIES,
SHARE YOUR DATA, DESIGNS,
FAILURES AND BEST PRACTICES AND
DUPLICATION OF WORK IS DEADLY.
I WANT TO THANK OUR MEMBERS TO
JOIN THE CONVERSATION AND WE'LL
HAVE EACH OF THEM INTRODUCE
THEMSELVES WE SHARING THEIR
CONNECTION TO ALS RESEARCH AND
THEN MOVE INTO A BRIEF
DISCUSSION PERIOD FOLLOWED BY
OUR QUESTION AND ANSWER PERIOD.
I'LL TURN IT TO DAN.
>>THANK YOU FOR HAVING ME.
I'M KULDIP AND I WAS AT THE
MICHAEL J. FOX FOUNDATION AND I
WORK THOR THE LARGEST PATIENT
ADVOCACY IN THE WORLD THE ALS
ASSOCIATION.
AND WE HAVE A CARE, RESEARCH AND
ADVOCACY APPROACH TO MAKE ALS A
LIVABLE DISEASE.
THAT MEANS LONGER LIVES,
LIFE-EXTENDING TREATMENTS FOR
PEOPLE WITH ALS AND IMPROVE
QUALITY OF LIVES AND GREATER
ENGAGEMENT AND AUTONOMY AND
REDUCING PHYSICAL EMOTIONAL
BURDENS AND FINANCIAL BURDENS
AND PROMOTING LOVED ONES FROM
GETTING ALS.
IF WE CAN FIND NEW RISK FACTORS
AND IDENTIFY THEM AND TREAT THEM
AS AS SOON AS POSSIBLE WE CAN
PREVENT NEW CASES OF ALS.
LISA ASKED TO SPEND TWO MINUTES
ON THOUGHT.
TWO THINGS COME TO MIND.
THERE WERE QUESTIONS AROUND ALS
RESEARCH NIH SHOULD SUPPORT AND
NIH PUT FORWARD 15 PRIORITIES IN
THE LAST TWO DAYS AND WHAT WE
NEED TO DO IS TO LOOK AT THE
MONEY EFFORT AND TIME TO IMPACT
THE 15 PRIORITY.
THAT'S WHAT NIH NEEDS NOW.
AND DEVELOPING A PLATFORM OF
TOOLS AND RESOURCES IS A
SIX-MONTHS TIME TO IMPACT.
HOW DO DO WE PRIORITIZE THE
PRIORITIES AND THE OTHER POINT I
WANT TO MAKE IS COLLABORATION
AND COORDINATION, I THINK WE
KIND OF GET THEM -- WE USE THEM
INTERCHANGEABLY.
THEY'RE NOT THE SAME THING.
COLLABORATION IS GETTING
EVERYBODY TO DO THE SAME THING
OR WORK IN THE SAME EXACT WAY.
COORDINATION IS KNOWING WHAT
EVERYBODY ELSE IS DOING AND THEN
TAKING THAT INTO ACCOUNT IN WHAT
YOU ARE WORKING ON.
THEY'RE BOTH IMPORTANT BUT WE
WANT DIVERSITY OF THOUGHT AND
APPROACH IN A SYSTEM AS COMPLEX
AS ALS.
THAT'S MY TWO POINTS OF VIEW.
>>THANK YOU.
KULDIP.
NETA.
>>I'M A NEURO SCIENTIST BY
TRAINING AND NOW YOU WORK IN
INDUSTRY IN PHARMACEUTICALS AND
I BRING A DIFFERENT PERSPECTIVE.
I WANT TO USE THIS PERSPECTIVE
TO BUILD ON WHAT KULDIP SAID.
ONE COMMON THING WE SEE ACROSS
THE DIFFERENT 15 PRIORITIES IS
THE SHARING OF DATA.
THE REASON FOR THIS IS THIS DATA
IS A PLATFORM OVER WHICH THE
EASIEST FOR US TO GROW BECAUSE
IN ORDER TO COLLABORATE, LIKE,
KULDIP SAID WE NEED TO AGREE ON
BASIC QUESTIONS AND IT'S NOT
ALWAYS EASY.
IN ORDER TO ALLOW DIVERSITY OF
APPROACHES WE WANT TO START BY
MAKING HIGH-QUALITY, LARGE DATA
SETS AVAILABLE FOR THE RESEARCH
COMMUNITY AND THERE BE ALLOWING
MANY DIFFERENT QUESTIONS TO BE
ANSWER IN MANY DIFFERENT WAYS.
THAT'S WHY WE CHOSE OUR FIRST
PRIORITY TO BE TO BRING TOGETHER
A LOT OF DATA.
THE THIRD PRIORITY FOR US AND
ANOTHER COMMON THEME IS HOW TO
BRING TOGETHER THE DIFFERENT
IDEAS AND LEARN ABOUT EACH
OTHER'S PRIORITIES SUCH THAT WE
CAN ALSO COLLABORATE
MEANINGFULLY HOW TO CREATE
MODELS TO UNDERSTAND HOW THE
DIFFERENT PERSPECTIVES COME
TOGETHER AND WHAT TYPE OF
QUESTIONS HELP US ADDRESS BOTH
THE SCIENCE AND THE TRANSLATION
OF THE SCIENCE INTO MEANINGFUL
DRUGS AND HOW TO INCORPORATE
MORE DIVERSITY OF PATIENTS AND
PATIENT'S EXPERIENCES INTO THAT
THINKING.
SO FIRST WE WANT TO CREATE A
FRAMEWORK THAT EVERYONE CAN USE
DIFFERENTLY FROM EACH OTHER AND
SECOND OF ALL WE WANT TO CREATE
A FRAMEWORK IN WHICH PEOPLE CAN
COME TOGETHER AND LEARN ABOUT
DIFFERENT APPROACHES AND
EVENTUALLY COME TO KEY QUESTIONS
TO BE ANSWERED TOGETHER.
>>THANK YOU.
WITHOUT THESE TWO COMBINED IT
WOULD BE HARD TO REACH THE
ULTIMATE GOAL WHICH IS TO ALSO
BRING TREATMENT FOR PATIENTS.
>>HI, I APOLOGIZE I'M AT A
FUNERAL AND IF I GO IN NOW, JUST
MOVE ON TO SOMEONE ELSE.
WHAT I WANT TO THANK BOTH OF YOU
FOR RUNNING THIS SECTION.
I'M A CLINICIAN AT JOHNS HOPKINS
AND I FOUNDED THE PACKARD CENTER
IN 2000 AND IT WAS DESIGNED TO
BE ABOUT COLLABORATION AND
BRINGING ACADEMICS TOGETHER TO
BRING TOGETHER MANDATORY
RESEARCH AND WE WERE FOCUSSED ON
TRYING TO GET THE EARLY
RESEARCH.
EARLY RESEARCH DRIVES THE FUTURE
DEVELOPMENT OF DRUGS.
AND I SHOULD MENTION BIOMARKERS
AS WELL.
THAT'S GONE ON BEAUTIFULLY FOR
23 YEARS.
IN 2015, WE CAME UP WITH A NEW
PLAN TO ADDRESS THE VERY CORE
ASPECT OF ALS WHICH IS STILL
UNTAPPED WHICH IS SPORADIC ALS
AND CAN WE IDENTIFY THE
BIOLOGICAL SUB GROUPS AND THAT
WAS THE PROGRAM I WAS THE
ARCHITECT FOR.
TODAY IT'S A COLLABORATION OF
100 RESEARCHERS AND 1,000
PATIENTS.
EVERYTHING WE'VE BEEN TALKING
ABOUT AND THOUGH I'LL CALL IT
SMALL SCALE IS LARGE AND WHAT WE
NEEDED WIN SHARING OF BIO FLUIDS
AND DATA AND AT-HOME MONITORING
OF PATIENTS TO GET THE DEEP DATA
WE GET WHEN PATIENTS CANT GET IT
TO CLINIC AND THE IPS CELLS
SERVING AS THE FOUNDATION TO
FINDING THE PATHOLOGICAL
PATHWAYS AND SPORADIC.
THERE'S NO SUCH THING AS AN A
MOUSE MODEL SPORADIC ALS.
I GREW UP WITH THOSE MODELS AND
KNOW THEM WELL AND SERVING THE
SPORADIC POPULATION.
AND ALL BUILDING THE
COLLABORATIVE TOOLS IS WHAT THIS
GROUP IS ABOUT AND NEED TO GET
TO OTHER POPULATIONS.
AND LASTLY, DAN CORRECTLY POINT
THE OUT HOW DO WE MAKE SURE THIS
IS SHARED WIDELY AND WE WERE
FORTUNATE TO PARTNER WITH
MICROSOFT AND BUILD A PORTAL
WHERE ALL THE DATA IS FREELY
SHARED AND TARGETED IMPORTANTLY
AND STRESSED WITH NO IP
RESTRICTIONS.
GIVE THE DATA OUT AND NO
UNIVERSITY CAN TRY TO OWN IT.
IT'S ALL FREELY AVAILABLE TO LET
THE WORLD'S COMMUNITY USE THAT
AS A STARTING POINT FOR
RESEARCH.
I THINK IT'S A SMALL EFFORT ON
OUR PART AND LARGE AMONG THE
GROUP WORKING ON IT AND THE IDEA
OF BRINGING OTHER RESOURCES THAT
ARE BEING DEVELOPED OR COULD BE
DEVELOPED ON THESE PLATFORMS TO
CHANGE THE COURSE OF OUR
UNDERSTANDING OF SPORADIC ALS
AND ALS IN GENERAL TO DISCOVER
EFFECTIVE THERAPIES.
>>THANKS, DAN AND LISA.
I'M ANDREA PAULS BACKMAN.
I LOST MY MOM THREE YEARS AFTER
HER DIAGNOSIS.
I WAS ONE OF HER PRIMARY
CAREGIVERS AND HONESTLY THE
PROCESS WAS SO PROFOUND IT
CHANGED MY LIFE IN MANY WAYS AND
CHANGED MY CAREER TRAJECTORY.
I LEFT A 30 YEAR CAREER IN
INVESTMENT MANAGEMENT AND
PRIVATE EQUITY AND GOT INVOLVED
IN THE ALS COMMUNITY.
I SERVED ON A BOARD OF TRUSTEES
MEMBER FOR THE ALS ASSOCIATION
AND I'VE BEEN THE CEO OF THE
OLDEST INDEPENDENT ALS
FOUNDATION IN THE COUNTRY AND ON
ASSOCIATIONS AND INVOLVED IN A
VARIETY OF INITIATIVES AND
ORGANIZATIONS.
I'M HAPPY TO BE PART OF THIS
GROUP.
COLLABORATION OF PARTNERSHIPS
ARE NEAR AND DEAR TO MY HEART
AND I WANT TO TOUCH ON THAT.
THERE'S LIMITED RESOURCES AND
TIME WHICH IS CLEARLY THE CASE
WHEN YOU'RE DEALING WITH A
TERMINAL, RAPIDLY PROGRESSING
DISEASE.
IN MY OWN EXPERIENCE, WHEN I
CAME TO LESS TURNER AND IN
WORKING WITH THE CENTER AT
WESTERN MEDICINE, I SAW IT HOW
IN MANY ACADEMIC INSTITUTIONS AN
OLDER MORE INSULATED MODEL WAS
HAMPERING THE SPEED OF
DISCOVERY.
THE WORK IS TOO IMPORTANT TO BE
DONE IN A VACUUM.
I COULD SEE THERE WERE FUNDERS
THAT WERE INCENTIVIZING THIS.
NOT DELIBERATELY BUT THAT WAS
THE DOWN STREAM EFFECT.
I LOOKED TO BECOME MORE
COLLABORATIVE AND WE PARTNERED
IN VARIOUS RESEARCH EFFORTS AND
EVERY OTHER ORGANIZATIONS ON
DRUG APPROVALS AND SEEING
IMPROVED RESULTS IN THE ALS
COMMUNE.
AND IN THE LESS TURNER
FOUNDATION WE WERE IN THE HOIMZ
OF PEOPLE LIVING WING ALS AND
GOT THEIR INPUT ON REAL TIME
BASIS.
AND WE NEED TO STRUCTURE IN THE
STRATEGIC PLAN.
>>THANK YOU.
ANDREA.
SHARON.
I'M SHARON HESTERLEE THE CHIEF
RESEARCH OFFICER AT THE MUSCULAR
DYSTROPHY ASSOCIATION.
I'VE BEEN IN THIS SPACE ABOUT 20
YEARS.
BOTH IN THE NONPROFIT SIDE AND
IN PHARMA AND BIOTECH COMPANIES
AND ALS IS A BIG PART OF OUR
PORTFOLIO AND ALWAYS HAS BEEN WE
WERE THE FIRST ORGANIZATION TO
FOCUS ON ALS RESEARCH AND HAVE
SPENT MILLIONS OVER THE YEARS
AND HAVE A DATABASE OF CLINICAL
DATA THAN HIGH QUALITY AN
ANONYMIZED DATA FROM OVER 2,000
PEOPLE PARTICIPANTS.
I KNOW MANY HAVE BEEN IN THE
SPACE AND IT'S MOSTLY
FRUSTRATING TO SEE HOW SLOW, HOW
MUCH EFFORTS HAS GONE INTO THIS
SPACE WITH WHAT FEELS LIKE
FRUSTRATINGLY SLOW OUTCOMES AND
IT'S CLEAR WE NEED TO DO
SOMETHING DIFFERENT TO START TO
SEE RESULTS.
IT'S A HARD DISEASE TO TACKLE.
THE COLLABORATIVE EFFORTS COULD
BE GAME CHANGERS FROM THAT
STANDPOINT FROM THE ONE THOUGHT
THEY HAVE TO BE PROPERLY
RESOURCED.
TO TAKE BIG AMBITIOUS PLANS AND
UNDER RESOURCE WILL SLOW US DOWN
BUT GOING FORWARD THE RIGHT
PLACE TO GO, WE JUST HAVE TO DO
IT RIGHT.
I'LL STOP THERE.
YOU CAN I'M HUMBLED ON A DAILY
BASIS BY THE PEOPLE I GET TO
INTERACT WITH AND I GOT ADVICE
EARLY ON THAT YOU HAVE TO LEAN
IN TO THE DISEASE AND I TRIED TO
DO THAT AND JEFF, YOU GOT FREE
ADVERTISING TODAY IN TERMS OF
THE PACKARD CENTER AND TIME
SPENT ON ALS AND I'M PASSIONATE
ABOUT THE ISSUE OF WHAT CELLS
CAN BRING TO THE FULL INDUSTRY
I'VE BEEN SURPRISED BY HOW
CONSISTENTLY OVER THE LAST TWO
DAYS THERE'S BEEN BROAD
CONSENSUS AROUND THE HYPOTHESIS
OF NOT ONE FORM OF ALS BUT SUB
GROUPS AND HOW CAN YOU DETERMINE
THIS WHEN YOU CAN'T TAKE
BIOPSIES LIKE CANCER AND USING
IPS CELLS ARE PART OF THE
ANSWER.
THERE ARE INITIATIVES UNDERWAY
SUCH AS ANSWER ALS TO DO THIS
BUT WE'RE PROPOSING TO BUILD OFF
THIS TO CREATE RICH RELATED DATA
AND BIO FLUID SAMPLES.
THIS NEED HAS BEEN HIGHLIGHTED
TIME AND TIME AGAIN BY THE OTHER
WORK GROUPS OVER THE PAST TWO
DAYS.
WHAT IF EVERY PATIENT ENTERING A
TRIAL YOU CONSISTENTLY CAPTURED
CLINICAL DATA IN THE GENOME
SEQUENCE AND BIO FLUID SAMPLES
AND MORE AND PUT IT ALL ON AN
OPEN SOURCE PLATFORM FOR
RESEARCHERS AS WELL AS THE
PHARMA COMMUNITY TO INTERROGATE
WITH A.I. AND MACHINE LEARNING
TECHNOLOGY.
SOME WILL SAY IT'S TOO
EXPENSIVE, HOWEVER, IS IT?
IF YOU COMPARE IT TO THE STATUS
QUO AND THE EXPENSE OF LOST
PRODUCTIVITY OF PATIENTS WHO DIE
EVERY YEAR, THE MASSIVE COST
INCURED BY THE FAMILIES,
CAREGIVERS AND HEALTH SYSTEMS TO
CARE FOR THOSE WHO SUFFER FROM
THE DISEASE AND THE COST
ACCUMULATED THROUGH THE YEARS BY
HUNDREDS OF FAILED DRUG TRIALS.
I'D ARGUE IT'S AN INVESTMENT WE
HAVE TO MAKE AND LOOK FORWARD TO
THE NIH LAYING OUT A PLAN TO
MOVING IT FORWARD FROM STRATEGY
AND ACTION.
IT'S A DIVERSE GROUP AND IT'S
RULE UNITED IN THESE THREE
PRIORITIES AS A WAY OF INVESTING
IN THE FIELD TO MOVE IT FORWARD.
>>WE'LL MOVE TO THE QUESTION
AND ANSWER PERIOD NOW AND START
WITH SOME QUESTIONS THAT WERE
RECEIVED YESTERDAY.
THE FIRST ONE IS TO JEFF AND
DAN.
HOW WILL YOUR PLANS FOR A ROBUST
ALS NATURAL HISTORY STUDY
LEVERAGE EXISTING NATURAL
HISTORY STUDIES?
>>THE GROUP DISCUSSED NATURAL
HISTORY.
OURS WAS FOCUSSED ON CLINICAL
DATA.
AND HOW DO WE INTEGRATE CLINIC
DATA SETS AND BIO BANKS.
THIS IS IMPORTANT.
YOU HEARD ABOUT ANSWER AND MAKE
SURE THE PATIENTS THAT ARE GOOD
ABOUT SUPPORTING CLINICAL
RESEARCH BENEFIT FROM THAT AND
HAS TO BE CONCERTED EFFORT TO
INTEGRATE DATA.
TO BE FAIR IT'S NOT LIKE MERGING
A BUNCH OF EXCEL SPREAD SHEET.
HOW DATA IS ANALYZED AND HOW YOU
CODE AND ONE PERSON CALLS A TIRE
AND ANOTHER PERSON CALLS A WHEEL
AND THEY ARE DIFFERENT ENTITIES.
THAT'S WORK PUTTING DATA
TOGETHER AND THAT THE NIH CAN
HELP TREMENDOUSLY ON.
THEY ALREADY HAVE EXISTING
DATABASE WHERE'S THEY INITIATED
THE INTEGRATION OF DATA AND
THERE'S BEEN EFFORT IN GETTING
DATA SETS FROM DIFFERENCE
SOURCES.
WE DON'T WANT TO DUPLICATE
EFFORTS, TRUE, BUT WE FIND WAYS
OF TAKING ADVANTAGE OF THE DATA
WOULD BE A HIGH PRIORITY BECAUSE
THEN YOU DON'T HAVE TO REPLICATE
THINGS.
THE COMMENT I DISAGREED WITH THE
REPLICATION OF EXPERIMENTS IS
CRITICAL.
SOMETIMES PEOPLE THINK, IF
SOMEONE MADE A DISCOVERY, WE
DON'T NEED TO DO IT AGAIN.
WHY WASTE THE MONEY.
SCIENCE IS ABOUT REPLICATION.
THE FIRST OBSERVATION IS STRONG
BUT IT COMES TO LARGE DATA SETS
AND INTEGRATING AND I THINK IT'S
POSSIBLE WITH THE HELP OF NIH.
>>THE NEXT QUESTION WHICH I
THINK HAS BEEN TOUCHED ON
BRIEFLY WE CAN PROBABLY KEEP THE
RESPONSE BRIEF, IN TRYING TO
ACCOMPLISH YOUR PRIORITIES, DO
YOU HAVE ENOUGH DATA OR DO YOU
NEED TO COLLECT MORE IF YOU NEED
TO COLLECT MORE HOW SPECIFICALLY
WILL YOU ENSURE YOUR DATA IS
AVAILABLE AND USABLE FROM OTHER
RESEARCHERS?
THIS IS ONE DAN OR JEFF CAN
ADDRESS.
>>WE NEED TO COLLECT A LOT MORE
DATA.
WITH THE DISEASE AS HETEROGENOUS
AS THIS ONE WITH DIFFERENT
PHENOTYPES WE NEED TO BE ABLE TO
IDENTIFY THEM AND THAT TAKES
DATA TO BE COLLECTED IN A VERY
CONSISTENT MANNER WITH
CONSISTENT PROTOCOLS.
AS WE'VE SAID MULTIPLE TIMES, WE
ALSO BELIEVE THAT DATA HAS TO BE
OPEN SOURCE.
IT HAS TO BE AVAILABLE BUT ALSO
HAS TO BE IN DATABASES THAT ARE
EASILY INREQUIRABLE SO PEOPLE
CAN ACTUALLY USE THEM
EFFECTIVELY.
>>LISA, CAN I ADD ONE QUICK
THING TO THAT.
I THINK NIH ALSO HAS TO FIND A
WAY TO INCENTIVIZE SCIENTIST TO
STUDY OR ANALYZE THAT DATA.
THERE'S A LOT OF RESOURCES AND
FUNDING TO PUT UP THIS TYPE OF
DATABASE THE SINGLE LARGEST
COLLECTION OF PATIENT DATA BUT
WE NEED PEOPLE TO ANALYZE THE
DATA.
IT'S EIGHT OF WORK AND WE WANT
TO MAKE SURE THAT ALL THAT
EFFORT IS NOT GOING WASTE.
WHETHER IT'S SCIENTIST OR A TEAM
AND DATA CHALLENGES, I THINK
THAT ANALYSIS NEEDS TO BE
INCENTIVIZED.
>>
>>I AGREE.
I KNEW THE NEXT PHASE IS WHO'S
GOING TO PAY THE MONEY TO USE
THE DATA AND NIH CAN PUT OUT AN
RFA SPECIFICALLY FOR USING
EXISTING DATA SETS AND OTHER
ORGANIZATIONS PUT TOGETHER A
LIST AND SAY COME TOGETHER WITH
EXPERIMENTS BASED ON USING SOME
OF THAT DATA RATHER THAN
WAITING.
AND DAN'S RIGHT, WE NEED MORE
DATA BUT THERE'S A LOT OF THAT
COULD BE INCREDIBLY USEFUL.
>>NOW WE'LL MOVE INTO A COUPLE
QUESTIONS THAT RELATE TO
AWARENESS OF ALS.
AND DAN, YOU WERE GOING TO TAKE
THOSE.
>>OKAY.
>>THE MONTH OF MAY IS ALS
AWARENESS MONTH.
IN 2022 BOTH CNN AND FOX NEWS
WEBSITES DON'T HAVE STORIES
ABOUT MAY BEING ALS AWARENESS
MONTH.
THE ORGANIZATIONS NEED TO MAKE A
GREATER EFFORT IN GETTING THE
WORD OUT.
I'VE RECEIVED LOTS OF E-MAILS
FROM ALS AND NEUROLOGICAL
ORGANIZATIONS ABOUT MAY BEING
ALS AWARENESS MONTH HOWEVER, THE
WRONG PEOPLE ARE BEING TARGETED.
PEOPLE WITH ALS ALREADY KNOW
ABOUT ALS.
AND THERE'S AAGE BEING SENT TO
THEM AND THANK YOU TO WHO RAISED
THE ISSUE.
IT'S A FRUSTRATING SITUATION AND
I AGREE 100% THAT FAMILIES
IMPACTED BY ALS SHOULD NOT BE
THE TARGET OF AWARENESS
CAMPAIGNS AND ONE THING IS THAT
IN MANY CASES IN ALL HONESTY, WE
MEAN FUND-RAISING.
WHICH IS AN EVEN STRONGER REASON
AS TO WHY THOSE IMPACTED BY ALS
SHOULD NOT BE TARGETED AND I'VE
ASKED THE QUESTION OF A FEW
ORGANIZATIONS.
WHY ARE WE NOT GOING OUT IN THE
WORLD TO SPREAD AWARENESS.
AND FROM WHAT I UNDERSTAND AND
THOUGHT NEEDS TO GO TO MAKE ALS
RELATABLE TO PEOPLE OUTSIDE OF
OUR COMMUNITY.
THEY WOULD NEED TO UNDERSTAND WE
LIKE TO SHOW THE EXTREMITY OF
THE DISEASE BECAUSE IT'S SO
EXTREME.
AND OTHERS CAN'T NECESSARILY
RELATE TO US.
WE BECOME THEN AND THEY HAVE
CAUSES AND THINGS THAT THEY DO
RELATE TO THAT DO IMPACT THEIR
FAMILIES SO THAT WOULD BE WHAT
THEY CHOOSE TO FOCUS ON.
TO ME, FUNDING PUTS IT TO A
JOINT COMMUNICATIONS CAMPAIGN
BETWEEN THE PUBLIC AND PRIVATE
SECTORS WOULD BE A POWERFUL
SPEND.
AND CAN YOU MAKE THE CAMPAIGN
WITH POP CELEBRITIYSS AND
INVESTORS AND I THINK I
UNDERSTAND THE BASIS.
WE WANT TO USE THE PUBLIC'S
ADORATION OF THOSE THEY KNOW
IMPACTED BY ALS IN ORDER TO MAKE
IT INTERESTING.
AND WHERE WOULD THEY DONATE AND
TO WHOM AND ARE YET TO HAVE A
LARGE ORGANIZED AWARENESS FUND
RAISING EFFORT BECAUSE THE TRUTH
IS MUCH OF THE WORK DONE IN OUR
COMMUNITY ORGANIZATION IS DONE
THROUGH FUNDING THAT'S BEEN O
ORGANIZED BY THAT ORGANIZATION
DIRECTLY.
AND PERHAPS YOU WANT TO EDUCATE
THE PUBLIC ABOUT THE DISEASE IS
A FAIR AND WORTHY CAUSE AND
SOMETHING ALL ORGANIZATIONS
CONTRIBUTE TO AND IT CAN ALSO
LEAD TO LARGE PUBLIC/PRIVATE
DONORS NOT NECESSARILY IN THE
PUBLIC EYE.
SHARON, DO YOU WANT TO ADD TO
THAT?
>>THAT WAS A PRETTY COMPLETE
RESPONSE AND THE THING THAT HAS
DONE MOST TO RAISE AWARENESS WAS
THE ICE BUCKET CHALLENGE.
IT GOT THE WORD OUT BUT I'M NOT
SURE PEOPLE UNDERSTOOD THE
DISEASE.
IF THERE'S SOME WAY TO PAIR THAT
AWARENESS WITH TRUE
UNDERSTANDING AND I THINK THE
POINT THAT WAS MADE WAS REALLY
GOOD THAT HAVING HAVING A
SPECIFIC COORDINATED GOAL FOR
WHICH YOU'RE RAISING FUNDS MIGHT
MOTIVATE THAT LARGE GIVING THAT
I THINK WE NEED TO SEE AND MAY
ATTRACT CELEBRITIES WHO MAY BE
DRAWN TO THE CAUSE.
HAVING THAT COORDINATED EFFORT
MAY HELP AND THE ICE BUCKET
CHALLENGE GOT US PART WAY THERE
AND NEED TO PAIR THAT WITH THIS
COORDINATED PLAN AND UNDERSTAND
THE LIVED EXPERIENCE OF ALS FOR
THE PUBLIC.
>>THANK YOU.
>>THIS QUESTION HAS TO DO IN
WITH FRAGMENTATION AND SILOING
OF THE RESEARCH WORK AND GOING
TO PROGRAMS THAT LACK THE
COLLABORATION.
WHAT ARE THOUGHTS ON HOW TO
ENCOURAGE ALS FUNDING
ORGANIZATIONS TO REQUIRE
INTERNATIONAL COLLABORATIONS AND
WHAT ARE THE BARRIERS FOR
COLLABORATIONS.
PERHAPS, ANDREA YOU CAN START
WITH YOUR THOUGHTS ON THAT.
>>THIS IS A BIG QUESTION AND I
KNOW ADAM AND JEFF WILL JUMP
INTO THIS.
I BELIEVE FUNDING ORGANIZATION
INCLUDING NIH ARE MOVING, NOT
UNIVERSALLY BUT MOVING THE
DIRECTION OF REQUIRING
COLLABORATION.
WE HAVE TO DEFINE WHAT THAT IS.
I WANT TO GET BACK TO THAT IN A
MOMENT.
THERE'S AN IMPORTANT NEED TO
HAVE OUTREACH INTO DIFFERENT
FIELD TO RESEARCH ALS.
I THINK DAN TALKED ABOUT THIS
EARLIER.
WE HAVE TO LOOK AT NATIONAL
COLLABORATION FIRST AND WE'LL
TALK ABOUT INTERNATIONAL AS
WELL.
AND I THINK THIS CAN BEST BE
ACCOMPLISHED BY SOMETHING WE
IDENTIFIED IN OUR THIRD PRIORITY
WHICH IS CREATING A
COMPREHENSIVE RESEARCH
LANDSCAPE.
SO THE POINT OF THAT WOULD BE TO
CAPTURE AND IDENTIFY ALL THE
AREAS IN WHICH RESEARCH IS
CURRENTLY TAKING PLACE.
NOT THAT RESEARCHERS DON'T KNOW
WHO'S LAB IS DOING WHAT WORK BUT
DO THE BIOTECH FIRMS AND V.C.
FIRMS DO THEY KNOW AS THEY DO
PRE-CLINICAL WORK WHO IS DOING
WORK IN DIFFERENT AREAS?
WE NEED A TRANSPARENCY INTO THE
PROCESS.
WHO'S DOING WORK AND WHAT AREAS
ARE THEY WORKING ON SO OTHER
RESEARCHERS AND FUNDERS CAN MOVE
FORWARD MORE EFFECTIVELY.
>>NIH GRANTS ARE BUILT AROUND
COLLABORATION AND THE NIH
RECENTLY PUT OUT GRANTS WHERE
YOU HAVE TO HAVE MULTIPLE
TECHNIQUES TOGETHER, NOT UNIQUE
TO ALS BUT THE CONCEPT OF
COLLABORATION HAS BEEN LONG
BUILT INTO MANY NIH AS THE MAJOR
FUNDER.
OF COURSE I BELIEVE WE SHOULD
ENCOURAGE COLLABORATION BUT THE
ORGANIZATION HAVE NOT BEEN
COLLABORATING WELL AND ALMOST
EVERYONE HERE ON THE GROUP FROM
DIFFERENT ORGANIZATIONS FOUND
WAYS TO COLLABORATE AND SHARE
CO-FUNDING MAKING MONEY GO
LONGER WHICH IS VERY IMPORTANT.
IT'S HARD TO MANDATE
COLLABORATION FOR A LOT OF
COMPANIES AND YET AND THERE'S
ALWAYS GOING TO BE OCCASIONAL
SILOS AND PHARMA WITH A
DIFFERENT FUNDING SOURCE AND
INTERESTED IN RELYING ON US TO
GET ACADEMICS AND TARGET HAS
DONE THAT AND I'D LOVE TO SEE
THAT EXPAND NOT TO TAKE AWAY
FROM TARGET BUT THEY HAVE TOOLS.
YOU'RE BRINGING IN A TINY
COMMUNITY TO THE BENCH OF
ACADEMICS WHERE DISCOVERIES
OCCUR.
IT'S IT'S ALL THE ORGANIZATIONS
AND THE NOVEL APPROACHES
ORGANIZATIONS CAN BRING TO THIS.
>>COLLABORATION IS NOT JUST A
CHALLENGE IN THE WORLD OF ALS
BUT EVERY BUSINESS.
I FOUND THROUGH THE YEARS THAT
GREAT INNOVATION COMES FROM
GREAT COLLABORATION AND IT'S NOT
THE PEOPLE ARE NOT GOOD PEOPLE
BUT IT'S HOW THEY'RE MEASURED.
THEY'RE TO BRING FUNDING BACK TO
THE INSTITUTION NOT MEASURED ON
WHAT THEY DO TO DRIVE
COLLABORATIVE EFFORTS ACROSS THE
COMMUNITY.
AND THEY MAKE IT A CONDITION OF
ENGAGEMENT.
JEFF TALKED ABOUT THE PACKARD
CENTER BUT WITH ANSWER ALS TO
PARTICIPATE YOU HAD TO AGREE TO
FOLLOW A COMMON PROCESS AND
AGREE TO PUT THE DATA ON OPEN
SOURCE PLATFORM.
YOU HAD TO AGREE YOU'D PUT THE
DATA OUT THERE WITH NO DELAY.
THE EXTERNAL WORLD GOT THE DATA
AS QUICKLY AS THE INTERNAL WORLD
AND PEOPLE IN THIS SPACE DO WANT
TO COLLABORATE AND THEY BOUGHT.
WE GOT 24 RESEARCH CENTER AND
100 P.I.s.
INHERENTLY THE PEOPLE IN THE
SPACE ARE WILLING TO DO THE
RIGHT THING AND WILLING TO
COLLABORATE.
THE FUNDERS, PACKARD CENTER,
TARGET, NIH, WE HAVE TO DO A
BETTER JOB OF MAKING
COLLABORATION A CONDITION EF THE
FUNDING.
>>ARE THERE ANY INITIATIVES OR
RECOMMENDATIONS SPECIFICALLY
ORIENTED TO HARNESS THE
CONTRIBUTION AND POTENTIATE THE
IMPACT OF RESEARCH IN COUNTRIES
AND I'M AFRAID WE MAY BE MISSING
IMPORTANT INFORMATION FROM
SOURCES OUTSIDE WEALTHIER
COUNTRIES.
I COULD NOT SEE A CLEAR
INDICATION OF THIS IN THE DRAFT
AND MAY NEED A SEPARATE FOCUS.
>>IF WE COULD HAVE TRIALS IN
PARTS PART OF THE WORLD IT WOULD
ACCELERATE TRIALS.
THERE'S A LOT OF TO DO ON THAT
FRONT.
WE BOTH ON THE INTERNATIONAL
ALLIANCE OF ALS AND AND
COMMITTEES AND THE INTERNATIONAL
ALLIANCE HAS BEEN TALKING
SERIOUSLY ABOUT DEVELOPING
INFRASTRUCTURE IN THAT UNDER
DEVELOPED AREAS AND THE IDEA IS
THAT EVEN THINGS LIKE COLLECTING
DNA SAMPLES TO WHOLE GENOME
SEQUENCING, SOME AREAS DON'T
HAVE THE PROTOCOLS IN PLACE.
OR MAY NOT HAVE MINUS 20
FREEZERS TO PUT THE DNA SAMPLES
IN LET ALONE INFRASTRUCTURE TO
DO THE ANALYSIS OF WHOLE GENOME
SEQUENCING AND SHARING THE DATA.
THERE'S A LOT TO BE DONE AND
INTERNATIONAL ALLIANCE AND
ORGANIZATIONS CAN TAKE THE
LEADERSHIP IN DRIVING
INFRASTRUCTURE ACROSS UNDER
REPRESENTED AREASES WE CAN
ACCELERATE RESEARCH.
>>WITH COUNT RIS AND INDIA HAVE
UNIQUE AND POWER DRUG THERAPY
LIKE TRADITIONAL CHINESE HERBAL
MEDICINE AND AYURVEDIC AND WHAT
PURSUIT HAS BEEN DONE WITH
COLLABORATIONS TO IDENTIFY
POTENTIAL THERAPIES?
THE NIH HAS PROGRAMS OPEN TO
INTERNATIONAL APPLICANTS SO
INVESTIGATORS ARE CHINA AND
INDIA ARE ALWAYS WELCOME TO
APPLY AND SOME OF THOSE IDEAS,
AYURVEDIC THERAPIES AND CURCUMIN
IS BEING TESTED NOW IN A TRIAL.
AGAIN, WE WANT ENGAGEMENT AND
IT'S A COMPLEX DISEASE.
WE WANT ENGAGEMENT FROM EVERY
PART OF THE WORLD.
NO MATTER WHERE THE GOOD IDEA
COMES FROM.
>>I'VE SEEN LITTLE EFFORT IN
THE U.S. RESEARCH SPACE TAKING
ADVANTAGE OF THAT.
THE CLOSEST COMES BACK TO THE
TARGETED ENGAGEMENT COMPANIES
AND THEY COME THROUGH US TO
DESIGN DRUG TRIALS BUT I'VE NOT
YET SEEN MUCH.
IF IT'S AN UNTAPPED OPPORTUNITY
WE SHOULD LOOK INTO IT.
WE HAD ONE MORE QUESTION.
>>THE QUESTION IS HOW DO YOU
FOSTER INDUSTRY, ACADEMIC, NON
PROFIT PARTNERSHIPS?
THE TARGET ALS MODEL.
WE TAKE INDUSTRY IN EVERYTHING
WE DO AND 50% OF OUR BOARD AND
OUR INDEPENDENT REVIEW COMMITTEE
WHICH MAKES EVERY DECISION ON
FUNDING.
THEY LED 50% OF THE CONSORTIA
WHICH HAS BEEN A SUCCESSFUL
MODEL AND FUNDED 41 CONSORTIA
AND SOME HAVE LED TO DRUG
DISCOVERY PROGRAMS BY
PHARMACEUTICAL OR BIO TECH
COMPANIES.
WE ACT AS A MATCH MAKER AND WE
STARTED AN INITIATIVE AND TALK
ABOUT BIOMARKERS AND HOW
IMPORTANT THEY WERE WAS TALKED
ABOUT YESTERDAY AND WE BROUGHT
TOGETHER 14 COMPANIES AND 10
ACADEMIC CENTERS, SIX ALS,
NONPROFITS AND TWO VENTURE
CAPITAL FIRMS TO DESIGN AN
EFFORT TO FOCUS ON BIOMARKERS A
COUPLE YEARS AGO AND WE MADE
TOGETHER A LOT OF PROGRESS.
THE KEY THING IS CLEAR DESIGN OF
PROGRAMS, LOW EGO WHICH IS
CRITICAL AND EFFECTIVE FUNDING
AND CLEAR SHARING OF INFORMATION
WITHIN THOSE CONSORTIA AND
PEOPLE ARE WILLING TO PLAY AND
IT'S A COMPELLING MODEL AND
OTHER ORGANIZATIONS HAVE DONE
THE SAME.
ONE THING WE SEE IN OTHER NIH
COLLABORATION AND THE AMP FOR
OTHER DISEASES AND OTHER ONES IS
THE NIH CAN BRING IN THE
REGULATORY PARTNERS WHICH ARE
MOURN STAKEHOLDERS AND BRING
TOGETHER ACADEMIC AND INDUSTRY
BECAUSE OF THE IMPORTANT ROLE
THEY PLAY IN THE OUTCOME OF ALL
THIS RESEARCH.
IF WE'RE GOING TO TALK ABOUT
LARGE DATABASES AND STORAGE,
I'VE BEEN DOING AND WHAT YOU
NEVER WANT TO LOSE ARE THOSE
VALUABLE RESOURCES THAT HAVE
BEEN BUILT UP OVER THE YEARS AND
THE AND IF NIH WAS WILLING TO
SAY WE'RE GOING TO HOUSE THIS
INFORMATION AND WE'RE GOING TOE
LOOK AT LONG-TERM REQUIRED
EFFORTS.
THIS IS WHERE NIH CAN BE VERY
VALUABLE IN HELPING US OUT.
>>HOW DO YOU ENSURE WE DON'T
BOIL THE OCEAN AND HAVE
QUARTERLY, QUANTIFIABLE
MEASURES?
THIS GETS TO UNDERSTANDING HOW
IS IT WE CAN MEASURE THE
PROCESS, WHAT METRICS ARE
IMPORTANT AND I THINK WE TALKED
A LITTLE BIT ABOUT GETTING
INFORMATION LIKE THAT FROM THE
ONGOING SUCCESSFUL
COLLABORATIONS.
HOW DO THEY DO IT AND WHAT ARE
THE KEY THINGS TO MEASURE AND
THE KEY QUESTION TO ASK.
I DON'T FLOW IF THERE'S SOMEBODY
ELSE ON THE COMMITTEE WHO WANTS
TO ANSWER THAT MORE COMPLETELY.
>>I'LL TAKE A MENTION.
WE'VE LAID A TREMENDOUS
CHALLENGE AT THE FEET OF THE NIH
AND WE HAVE 15 INITIATIVES, LOTS
OF GREAT THOUGHTS IN TERMS OF
HOW YOU ADVANCE THE SCIENCE IN
THE SPACE.
I THINK THE CHALLENGE FOR THE
NIH IS TO BOIL THAT DOWN AND
DETERMINE WHAT ARE THE TWO TO
THREE MAYBE FOUR KEY ENABLERS
THAT WILL HAVE A HUGE IMPACT ON
THE SCIENCE.
AND THEN FOR EACH OF THOSE, YOU
GET THE CLEAR SCOPE DELIVERABLE
RESOURCE AND THE GOVERNANCE
PROCESS THAT DRIVES PACE AND
CADENCE AND EXECUTION.
THERE'S THE ONLY WAY TO ADVANCE
THINGS AND BACK TO THE QUESTION
NOT BOIL THE OCEAN BUT ADVANCE
THOSE CRITICAL THINGS THAT ARE
GOING TO MAKE THE BIGGEST
DIFFERENCE.
THAT WOULD BE MY VIEW.
THANK YOU TO THE AMAZING GROUP
AND THE ATTENDEES FOR SUCH GREAT
QUESTIONS WE'LL CONTINUE TO
COLLECT QUESTIONS ON THE MEDIA
WEBSITE AND WE LOOK FORWARD TO
CONTINUING TO SHAPE THE ALS
STRATEGIC PLAN AS YOUR EXCELLENT
QUESTION SHEDS LIGHT ON
IMPORTANT OPPORTUNITIES TO
ADVANCE ALS RESEARCH.
THANK YOU AGAIN.
>>I WANT TO THANK EVERYONE WHO
PARTICIPATED IN THIS DISCUSSION
AND DIALOGUE.
VERY INSTRUCTIVE AND WILL GUIDE
US GOING FORWARD.
WE'LL NOW TAKE A SHORT BREAK.
THE WORKSHOP WILL RESUME 2:15
AND WHEN WE COME BACK WE'LL
START THE PUBLIC EXCELLENT
SESSION.
YOU'RE WELCOME TO LEAVE THE
WORKSHOP RUNNING OR LOG OUT AND
BACK IN.
EITHER WAY WE'LL SEE YOU SOON.
THANK YOU.
>>WELCOME BACK AND WHAT
WE'D LIKE TO DO SIMILAR TO
YESTERDAY IS OPEN FOR COMMENTS.
YESTERDAY WE WERE READING A LOT
OF COMMENTS THAT WERE COMING IN
FROM THE AUDIENCE AND THEY WERE
EITHER POSTED EARLIER OR THEY
CAN ALSO BE POSTED IN THE Q&A IF
YOU HAVE ANY COMMENTS YOU STILL
WANT TO PROVIDE US WITH THAT YOU
WANT US TO SHARE WITH THE
COMMUNITY AND THE ATTENDEES OF
TODAY'S SESSION.
I WELCOME YOU TO PUT THE
COMMENTS OUT AND WE'LL READ THEM
OUT LOUD AND IF WE HAVE EXTRA
TIME WE'LL MAKE AN ATTEMPT TO
ANSWER THE UNANSWERED QUESTIONS
ACCUMULATED OVER THE LAST TWO
DAYS AND THERE WERE MANY
FANTASTIC QUESTION AND WE'LL SEE
IF WE HAVE TIME TO ADDRESS THOSE
QUESTIONS AND HAVE SOME OF THE
PANELISTS ANSWER THEM DIRECTLY.
IF WE CAN HAVE THE FIRST
COMMENTS.
I'LL HAVE YOU START WITH THE
FIRST COMMENT.
>>THIS IS FROM TIM LOWRY.
I HAVE BEEN LIVING WITH ALS
SINCE 2018 AND LEARNED MUCH FROM
MANY PARTICIPATING IN THE ALS
STRATEGIC PLAN, THANK YOU, FOR
YOUR RE LENTLESS WORK AND
THERE'S MUCH TO BE EXCITED
ABOUT.
THANK YOU.
>>THE NEXT COMMENT IS
ANONYMOUS, IF I MADE THE XHOIS
TO KNOW MY GENETIC STATUS IT'S
OFFICE TO BE ABLE TO ACT FOR MY
DESCENDANTS AND MYSELF BY
TESTING DRUGS BEFORE BEING SICK
AND IT'S DWE PLORABLE THAT
NOTHING IS ATTEMPTED FOR PEOPLE
WHO HAVE A HIGH PROB AT OF --
PROBABILITY OF BEING SICK, SO
YES, TRIPLE YES, TO TRY
EVERYTHING FOR EVERYTHING.
THANK YOU.
>>THIS STATES, I ADVOCATE THE
FURTHER FUNDING OF
PRE-SYMPTOMATIC C9 ORF72
TREATMENT SUCH AS CRISPR AND
OTHER SOLUTION CAN MORE FUNDING
AND STRATEGY BE PLACED INTO THIS
LIFE-SAVING TREATMENT.
>>THE NEXT COMMENT IS FROM GLEN
ROUSE.
I WAS DIAGNOSED IN SEPTEMBER OF
2020 AND I WAS ABLE TO DRIVE
MYSELF TO THE CLINIC IN
SACRAMENTO WHICH WAS TWO AND A
HALF HOURS AWAY.
I WAS ALSO ON THE PLATFORM FILE
REGIMENT B FOR VERDIPERSTAT IT
DOES NOT MEET THE STATISTICAL
END POINT AND CONTACTED BY THE
RESEARCH COORDINATOR WHERE I DID
THE TRIAL AND NEEDED TO HAVE MY
LAST VISIT TO FINISH IT AND SAN
FRANCISCO WAS THREE HOURS AWAY.
>>WALTER, THERE'S A BIT MORE IN
THE COMMENT.
>>I'M SORRY.
>>HERE WE GO.
ONE THING I DID NOT HEAR
YESTERDAY IN LIGHT OF THE FOCUS
GROUP IN THE CLINICAL CARE, DUE
TO MY PROGRESSION, IF I HAVE TO
ATTEND THE CLINIC OR GO TO SAN
FRANCISCO I CAN NO LONGER DRIVE
MYSELF OR RIDE IN THE CAR.
IN ORDER TO GO TO THE
APPOINTMENTS I HAVE TO BE
TRANSPORTED BY A MEDICAL VAN.
THIS IS A TREMENDOUS EXPENSE FOR
MYSELF.
GOING THROUGH MY ALS COURSE COST
$1,900 PER HOUR FOR AS LONG AS
THE CLINIC TAKES.
I MENTION THIS BECAUSE THE
STRATEGIC PLAN SHOULD ADDRESS
HOW TO ACCOMMODATE PEOPLE WITH
ALS AS THE DISEASE PROGRESSES
THAT WOULD SEEM SOME SORT OF
VIRTUAL VISIT OR ZOOM CALL WOULD
BE ONE WAY TO HANDLE THIS
PROBLEM AND ALLOW ME TO
PARTICIPATE MORE EASILY.
>>WE'LL HAVE A COMMENT FROM
PEGGY JOHNSON.
>>MY FATHER DIED 30 YEARS AGO
AND MY MOM DIED OF ALS.
IN THAT TIME, THE ADVANCEMENT
AND AVAILABILITY OF MEDICATION
FOR THE OTHER POPULATION WITH
OTHER POPULATIONS HAS FAR
SURPASSED ANYTHING DONE WITH ALS
PATIENTS.
THERE'S MONEY AND IT NEEDS TO BE
USED TO GETTING ACCESS TO
WHATEVER EXPERIMENTAL TRACKS
HAVE A BASELINE OF DATA CHOSEN
FOR THE PATIENT FOR THEIR
TREATMENT PLAN.
THANK YOU.
>>NEXT IS A COMMENT, I AM
CONFUSED, DISGUSTED AND OUTRAGED
AS A TAXPAYER AND ALS GENE
CARRIER THAT ALS PATIENTS AND
GENE CARRIERS CAN'T AFFORD OR
HAVE ACCESS TO DRUGS THAT WOULD
GIVE THEM HOPE, IMPROVE AND
EXTEND THEIR LIVES.
THROUGH THE OUTRAGE THAT I CARRY
THE GENE FOR A DEVASTATING
DISEASE MAY HAVE NO HOPE FOR
PREVENTING IT TREATING IT, BEING
CARED FOR, CURING IT, SURVIVING
IT OR EVEN DYING WITH DIGNITY.
>>THANK YOU.
>>THE NEXT COMMENT COMES FROM
MAGGIE WHO SAYS MY MOTHER DIED
WAITING FOR HELP AND SHE
SUFFERED.
I SUFFERED AS HER PRIMARY
CAREGIVER.
IT WAS DEVASTATING AND HELLISH
AND NOW I'M CARRYING THE GENE.
GIVE ME HELP, PLEASE.
THANK YOU, MAGGIE.
WE HEAR YOU AND THAT'S EXACTLY
WHAT WE WANT TO DO.
IN 2012 WE LAUNCH THE ALS
EMERGENCY TREATMENT FUND WHICH
WAS THE FIRST ORGANIZATION IN
ANY THERAPEUTIC AREA NOT JUST
ALSO TO FOCUS ON LARGE SCALABLE
EXPANDED ACCESS PROGRAMS
FEASIBLE FOR DRUG DEVELOPERS AND
HEALTH CARE PROVIDER.
THE ORGANIZATION EXISTED FROM
2012 THROUGH 2018 DURING WHICH
TIME IT AUTHORED THE EXPANDED
ACCESS SESSION FOR THE FDA
GUIDANCE ON ALS DRUG DEVELOPMENT
AND LATER EXPANDED THE LANGUAGE
OF THE REAUTHORIZATION ACT OF
2016 SECTION 610 WHICH HAS
REPRESENTATIVE NESS AND
INCLUSIVITY AND THERE WAS A
PLATFORM FOR LARGE COHORT
EXPANDED ACCESS PROGRAMS SERVING
AS TREATMENT USE TRIALS FOR ALL
SEEKING NEW TREATMENT OPTIONS
AND FOR VALID REASONS ARE NOT
ABLE TO PARTICIPATE IN THE
RESEARCH TRIALS.
FIRST THE SPONSORS HAD COST
BURDEN FOR PATIENT AND THE
LIMITED TIME AND RESOURCES OF
PHYSICIANS INCLUDING THOSE AT
NON-RESEARCH CLINICS WHO WOULD
LIKE TO BRING INVESTIGATIONAL
TREATMENT AND DIAGNOSTIC OPTIONS
TO THEIR PATIENTS.
THE DRAFT AUTHORS SHOULD BE
COMMENDED FOR REFERENCING
EXPANDED ACCESS FIRST AS A
CAPABILITY OF THE CLINIC CORE
AND SECOND AS A FACET OF THE
PATIENT CENTRICITY BUT THE DRAFT
DOES NOT INCLUDE A PLAN FOR
MAKING IT FEASIBLE AT SCALE FOR
THE MEANINGFUL PROPORTION OF THE
DISEASE POPULATION.
NOR DOES IT CITE THE DISCOVERIES
MADE FROM EXPANDED PROGRAMS AND
OTHER THERAPEUTIC AREAS WHICH
WOULD NOT HAVE BEEN ACHIEVED
FROM RESEARCH TRIALS ALONE.
A PRIORITY SHOULD BE TO
INTEGRATE EDUCATIONAL
THERMOMETERS INTO A BROADER
RANGE OF CARE SETTINGS AND
CLINICS AND PROVIDERS THROUGH
EXPANDED ACCESS PROGRAMS THAT
ARE NEITHER COSTLY TO COMPANIES
AND UNDULY BURDENSOME.
NOW MAY BE A BETTER TIME THAN
WHEN WE FIRST PROPOSED IT DUE TO
THE WAVE OF THE SOLUTIONS IN E
PRO AND E-CONSENT
INTEROPERABILITY BETWEEN SYSTEMS
AND THE GROWING RECOGNITION OF
INVESTIGATIONAL TREATMENT AS A
LEGITIMATE CARE OPTION INSIDE
AND OUTSIDE THE TRADITIONAL
CLINICAL TRIAL ENTERPRISE.
THANK YOU.
>>WE HAVE ANOTHER COMMENT FROM
MIKE.
>>I HAVE TWO COMMENTS.
FIRST IS ON THE PRIORITIZATION
OF THE STRATEGIC PLAN.
I BELIEVE ACCELERATING THE
RESEARCH SHOULD BE OUR NUMBER
ONE PRIORITY WITH IDENTIFYING
OPPORTUNITIES FOR COLLABORATION
AND PARTNERSHIPS.
I BELIEVE IT WILL GIVE US
TARGETS FOR THERAPIES AND THE
RESEARCH AND OPTIMIZING THE ALS
CLVRJ RESEARCH IS THE NEXT
PRIORITY FOLLOWED BY THE QUALITY
OF LIFE.
I FEEL LIKE THE INDUSTRY WITH
THE $60,000 WE'RE PAYING FOR ALS
WHEELCHAIRS, THERE'S ENOUGH TO
MONEY PRIVATE COMPANIES CAN
HANDLE THAT.
MY SECOND COMMENT IS USEFUL
ASSETS.
WE LIKE TO LEVERAGE BIO
REPOSITORIES AND OTHER
REPOSITORIES.
TREMENDOUS AMOUNTS OF EFFORTS
AND DOLLARS HAVE BEEN INVESTED
IN CREATING THESE.
LEVERAGING THE EXISTING ASSETS
AND VALUE IS IMPORTANT TO THOSE
STAKEHOLDERS AND IMAGINE PUTTING
A CAR TOGETHER MANUFACTURED AT
SILOED PARTS AND THIS IS FROM A
SON WHERE JOHNNY CASH SUNG ABOUT
A CAR FROM PARTS ACQUIRED OVER
20 YEARS AND THIS IS THE SAME
IDEA AND AS MUCH AS WE'D LIKE TO
THERE'S SOME FACETS THAT AREN'T
WIDELY USABLE.
ORGANIZATIONS THAT HAVE THE
ASSETS SEEM TO REQUIRE THEM TO
BE USED AS A CONDITION FOR
COLLABORATION.
IN MY EXPERIENCE REQUIRING THE
ASSETS IS A BARRIER TO
COLLABORATION.
WE ATTEMPTED TO USE THOSE ASSETS
AND WE MAY BUILD THE SAME
CADILLAC WITH DIFFERENT PARTS
AND EACH MY HAVE A DIFFERENT
ASSET FROM THE RESEARCH DESIGN
AND WE NEED TO COLLECT IT
PERSPECTIVIVELY.
THE SAMPLE OF THE REPOSITORY
MISSING FUNCTIONAL RATES OF
DECLINE.
THIS IS CHALLENGING GOING
FORWARD BUT CHANGING WITH SAMPLE
COLLECTION PROGRAMS.
I'LL ENCOURAGE THE ALS FUNDING
ORGANIZATIONS TO LOOK OVERCOME
THE COLLABORATION BARRIER.
THANK YOU.
>>TO THE PRE-SUBMITTED
COMMENTS.
ONE PERSON SAYS MY FATHER WAS
DIAGNOSED WITH ALS AND FOUND TO
HAVE THE C9 GENE MUTATION.
WHILE HE'S THE FIRST IN MY
FAMILY TO BE DIAGNOSED WITH ALS
WE KNOW THE GENE IS INHERITABLE
AND I TESTED POSITIVE FOR THE
SAME MUTATION AND IT'S A
POSSIBILITY TO PASS IT DOWN TO
MY FUTURE CHILDREN.
I'M GRATEFUL FOR THE NINDS FOR
FORMING THIS WORKING GROUP AND
DEVELOPING THE STRATEGIC PLAN.
HOWEVER, MORE NEEDS TO BE DONE.
THERE NEEDS TO BE MORE RESEARCH
IN THE PRE-SYMPTOMATIC GENE
CARRIERS LIKE MYSELF TO HAVE
ACCESS TO TREATMENTS THAT WOULD
PREVENT THE SYMPTOMS TO MANI
MANIFEST.
WHEN MY FATHER RESOURCED HIS
GENETIC TESTING RESULTS I WAS
BASICALLY ON MY OWN TO DETERMINE
WHAT TO DO ABOUT MY OWN TESTING
AND TREATMENT.
THERE NEEDS TO BE MORE SUPPORT
FOR THOSE WHO ARE AT RISK OF
DEVELOPING ALS.
RECENT GENE CARRIERS NEED TO BE
INCLUDED IN THE STUDIES TESTING
POTENTIAL TREATMENT AS IT'S THE
ONLY WAY TO KNOW IF ALS CAN BE
PREVENTED OR EVEN CURED.
RIGHT NOW, THERE'S NO BEST
EVIDENCE FOR WHAT I CAN DO TO
PREVENT THIS DISEASE FROM M
MANIFESTING AND LIKELY TOO LATE
FOR MY FATHER TO BE ENROLLED IN
ABE STUDIES TESTING NEW
THERAPIES.
MORE NEEDS TO BE DONE FOR US.
WE NEED ACCESS TO POTENTIAL
THERAPIES AT THE SAME RATE OF
THOSE LIVING WITH ALS.
THANK YOU.
>>THIS IS FROM PETE SENSE
SPENCER.
I LOST A FRIEND AND THANK YOU
FOR THE OPPORTUNITY TO DEVELOP
TO THE NEW STRATEGIC PLAN.
THERE'S SO MUCH GOOD I SEE IN
THE DRAFT I WANT TO EMPHASIZE
THE IMPORTANCE OF ONE THEME
RUNNING THROUGHOUT THE PLAN ALL
FIVE WORKING GROUPS AT SOME
POINT IDENTIFIED THE NEED TO
GATHER MORE DATA FROM A
POPULATION OF PEOPLE LIVING WITH
ALS.
I STRONGLY SUPPORT THE MORE OF
REACHING MORE UNDER REPRESENTED
PEOPLE AND DOCTORS FOR INCLUSION
IN RESEARCH.
MANY STATED THE SCIENTIFIC VALUE
BUT ALSO SIMPLY THE RIGHT THING
TO DO.
NO ONE SHOULD BE PRECLUDED FROM
PARTICIPATING IN RESEARCH VIA A
NATURAL HISTORY STUDY OR
CLINICAL TRIAL BECAUSE THEY DO
NOT HAVE THE MEANS TO GET TO AN
ALS CENTER OF EXCELLENCE OR ALS
CLINIC.
I OFTEN WONDER ABOUT THE PEOPLE
WITH ALS I DO NOT SEE.
ALMOST ALL THE TERRIFIC PEOPLE I
HAVE MET IN THE ALS ADVOCACY
COMMUNITY ARE PROFESSIONALS WITH
AT LEAST ONE POST SECONDARY
DEGREE AND WHILE IT TOOK SOME
TIME THEY'RE NOW CONNECTED TO
OTHERS AND AWARE OF ALS
RESEARCH, TREATMENTS AND
CLINICAL TRIALS.
HOWEVER, MORE THAN HALF OF ALL
AMERICANS HAVE NOT COMPLETED A
COLLEGE DEGREE AND I SEE FEW OF
THEM REPRESENTED IN ALS ADVOCACY
GROUPS OR ON SOCIAL MEDIA.
THEY MAY BE LESS ABLE TO
SELF-ADVOCATE AND LESS AWARE OF
TREATMENT OPTIONS.
THERE ARE IT'S CRITICAL TO
EXPAND AWARENESS, TRAINING AND
PARTICIPATION IN ALS RESEARCH TO
PRIMARY CARE PHYSICIANS AND
NEUROLOGISTS IN ALL AREAS OF THE
COUNTRY SO ALL PEOPLE WITH ALS
WILL BE ENCOURAGED AND ABLE TO
PARTICIPATE.
THE ALS ADVOCACY COMMUNITY IS
WILLING AND READY TO HELP WITH
THIS.
THANK YOU, PETE.
>>
>>THE KEY TO EVERYTHING FOR A
PERSON LIVING WITH ALS IS AN ALS
DIAGNOSIS.
WITHOUT IT THERE ARE NO DISEASE
MODIFYING MEDICATIONS,
EQUIPMENT, SUPPORT, CARE
COORDINATION, ACCESS TO CLINICAL
TRIALS OR GENETIC TESTING.
CERTAIN GROUPS OF PEOPLE
EXPERIENCE DELAYS SUCH AS BLACK
PATIENTS EXPERIENCE AN
ADDITIONAL 6 TO 8 MONTH DELAY ON
TOP OF THE AVERAGE 12 MONTH
DELAY FOR SYMPTOM ONSET FOR ALS
PATIENTS.
IN MANY CASES THIS IS NOT ONLY
INCREDIBLY DIFFICULT AND
ISOLATING FOR THE PATIENT BUT
MAY ALSO BE LIFE-THREATENING AND
THE IDEAL WINDOW TO SLOW THE
PROGRESSION AND GAIN ACCESS TO
CLINICAL TRIALS MAY BE MISSED.
IT'S IMPORTANT THAT THE
STRUCTURE AND SYSTEMIC BARRIERS
THAT DISADVANTAGED CERTAIN
POPULATIONS FROM RECEIVING A
DIAGNOSIS ARE ADDRESSED IN THE
IMPLEMENTATION OF THE ALS PLAN
TO BE IMPACTFUL AND MEANINGFUL.
THANK YOU.
>>THERE'S A FEW MORE COMMENTS
THAT I'M PULLING OUT FROM THE
CHAT.
IT WAS CLEAR FROM THE TWO DAYS
THE PATH FORWARD IS FOR ALL
RESEARCHERS TO HAVE ACCESS TO
BROAD AND INCLUSIVE DATA SET
THAT INCLUDES A LARGE POPULATION
OF ALS PATIENTS, CLINICAL,
GENETIC AND BIO FLUID AND TISSUE
SAMPLES WITH WHICH TO CONDUCT
THEIR RESEARCH.
THIS WILL CLEARLY INCENTIVIZE
RESEARCH AND FOCUS THE RESEARCH
ON THE KEY KNOWLEDGE GAPS THAT
ONCE FILLED WILL LEAD TO
EFFECTIVE THERAPIES AND MORE
STREAMLINE AND EFFICIENT
CLINICAL TRIAL PROCESS.
IT'S BEEN DONE FOR CANCER AND
HIV NOW IT'S TIME TO FOCUS THE
BUDGET AND RESOURCES TO FOCUS ON
ALS.
FOR YEARS, ALS PATIENTS AND
THEIR FAMILIES HAVE BEEN THE
BETTER OF THE CASINO.
IT'S TIME FOR OUR COMMUNITY TO
BECOME THE HOUSE.
>>AFTER READING THE DRAFT I SEE
THE BUILDING BLACK RESONATES
THROUGHOUT.
I HOPE AS THE PLAN IS FINALIZED
A ROBUST NIH JUMPS TO THE TOP OF
THE LIST.
THIS IS NEEDED TO CONTRIBUTE TO
BUILD A BETTER ALS RESEARCH
COMMUNITY.
>>THERE WAS ONE MORE COMMENT
AND ASYSTEMATIC GENE CARRIERS
DON'T NEED TO PONDER.
WE WON'T BE IGNORED ANYMORE.
THANK YOU AND FOR EVERYBODY ELSE
WHO LEFT COMMENTS THAT WE WERE
HAPPY TO SHARE WITH EVERYBODY.
WE HAVE A LITTLE BIT MER TIME
AND WE WERE HOPING TO GET TO
QUESTIONS THAT WERE LEFT
UNANSWERED AND ONE WAS TO OUR
TRANSLATIONAL RESEARCH WORKING
GROUP I THINK NEIL SCHNEIDER
WANTED TO ADDRESS ONE QUESTION.
DO YOU HAVE THE QUESTION READY?
>>I'M PLEASED TO HAVE THE
OPPORTUNITY.
A NUMBER OF QUESTIONS CAME UP
YESTERDAY RELATED TO OUR FIRST
PRIORITY TO ESTABLISH CENTERS OF
EXCELLENCE PROGRAM IN ALS.
THERE WERE A NUMBER OF QUESTIONS
WITH RELATED CONCERNS.
ONE QUESTION READ CENTERS OF
EXCELLENCE ARE HELPFUL IN
PROVIDING INFRASTRUCTURE AND
INTERACTION AND SHARED RESOURCES
AND SUPPORT FOR NEW
INVESTIGATORS, ETCETERA.
MEASURES ARE NEEDED TO ASSURE
CONTINUED QUALITY AND
COMPETITIVENESS.
WHAT KIND OF MEASURES WOULD
ENSURE THESE CENTERS CONTINUE TO
CONDUCT HIGH-QUALITY, CUTTING
EDGE RESEARCH AND SERVE THE
BROADER RESEARCH COMMUNITY.
>>I LIKE THIS QUESTION AND MOST
THE RELATED QUESTIONS BECAUSE IT
FIRST ACKNOWLEDGES THE
IMPORTANCE OF THE ADVANTAGES OF
A CENTER IN EXCELLENCE PROGRAM
AND SHARED RESOURCES AND SUPPORT
FOR INVESTIGATORS.
THERE'S CONCERN EXPRESSED THAT
THESE CENTERS OF EXCELLENCE MAY
CONCENTRATE LIMITED RESOURCES TO
THE HANDS OF A SMALL NUMBER OF
LARGE ACADEMIC CENTERS LIKE THE
ONE THAT I LEAD AT THE EXPENSE
OF PATIENTS WHO DO NOT HAVE
ACCESS TO THESE LARGE ACADEMIC
CENTERS AS EXACERBATING WHO GETS
CARED FOR AND WHO DOES NOT.
THAT WAS NOT OUR INTENTION.
ON THE CONTRARY, WHAT WE THINK
IS NECESSARY AS WE'VE TALKED
ABOUT FOR TWO DAYS WE'VE TALKED
ABOUT BUILDING AN INFRASTRUCTURE
TO SUPPORT BASIC SCIENCE AND
TRANSLATIONAL SCIENCE AND
CLINICAL RESEARCH IN ALS AND A
LOT OF WHAT IS BEING SUGGESTED
TO THE NINDS IS IS A LOT OF WORK
AND EFFORT AND WHO WILL DO THE
WORK AND EFFORT AND WHAT IS
NEEDED TO DO IT.
WHATEVER THE NINDS DECIDES TO DO
WITH OUR RECOMMENDATIONS.
I THINK WHAT WE'RE TRYING DO FOR
CENTERS OF EXCELLENCE PROGRAM IS
TO BEGIN TO BUILD THE
INFRASTRUCTURE AND PROVIDE
SUPPORT FOR THE PROGRAMS THAT
ARE NEEDED TO MOVE US FORWARD.
WE'RE NOT TRYING TO GIVE
ADDITIONAL RESOURCE TO PEOPLE
WHO HAVE ENOUGH.
WE'RE TRYING TO ESTABLISH A
LARGER NETWORK OF CENTERS THAT
WOULD INVOLVE MORE PATIENTS SO
THE NODES SERVE AS AN ORGANIZING
CENTER FOR OTHER CENTERS IN
REGIONS OF THE COUNTRY THAT CAN
FEED PATIENTS TO THESE CENTERS
TO REACH OUT TO THEM REMOTELY
THROUGH DIGITAL TECHNOLOGIES AND
REMOTE CONSENTING, WORKING IN
THE FIELD TO INVOLVE AS MANY AS
PATIENTS AS POSSIBLE IN THE
IMPORTANT WORK WE HAVE TO DO.
AND WHAT IS ASKED IS IMPORTANT
AND QUALITY MEASURES ARE NEEDED
TO ASSURE CONTINUED QUALITY
COMPETITIVENESS AND WHAT KIND OF
MEASURES WOULD ENSURE THE
CENTERS CONTINUE TO BE HIGH
QUALITY.
THERE'S EXAMPLES OF THESE
CENTERS OF EXCELLENCE AND THE
CENTERS IN MUSCULAR DYSTROPHY
AND THE UDALL CENTERS AND
THERE'S EXAMPLES THAT WORK WEM
IN DIFFERENT WAYS.
WE DIDN'T WANT TO BE TOO
PRESCRIPTIVE TO WHAT THE PROGRAM
WOULD LOOK LIKE BECAUSE THERE'S
DIFFERENT MODELS FOR WHAT MIGHT
WORK AND THERE'S DECISIONS TO BE
MADE AS THERE ARE THE PRIORITIES
AND HOW IT WILL BE IMPLEMENTED.
WE HAD IDEAS WE SUBMITTED.
CORES OF THE CENTERS AND METRICS
THAT NEED TO BE MET AND PEOPLE
NEED TO SHOW THEY MET THE
MILESTONES THEY SET OUT IN
ADVANCE AND HAVE SUCCEEDED AND
WHAT THEY LAID OUT AND THIS
CENTERS HAVE TO UNDER GO
COMPETITIVE RENEWAL ON A TYPICAL
5-YEAR TERM.
I THINK THE CENTERS HAVE AN
IMPORTANT ROLE TO PLAY IN
ESTABLISHING THE STRUCTURE
NEEDED TO DO THE WORK WE'VE BEEN
TALKING ABOUT FOR TWO DAYS AND
WHAT THE CENTERS LOOK LIKE AND
HOW MANY THERE ARE AND WHERE
THEY'RE PLACED IS A DECISION FOR
THE NINDS.
>>I APPRECIATE YOUR
RE-ADDRESSING THE QUESTION THAT
CAME UP.
I THINK THIS IS IT FOR THIS
SESSION.
WE DON'T HAVE ANY ADDITIONAL
COMMENTS OR QUESTIONS THAT WE
HAVE TO ADDRESS.
SO CERTAINLY FOR THOSE OF YOU
WHO HAD OTHER QUESTIONS THAT
CAME UP YESTERDAY AND TODAY,
HOPEFULLY ALL THE QUESTIONS WERE
ADDRESSED EITHER DURING THE
SESSION SLIDE OR BY THE NIH
STAFF THAT FEVERISHLY TRIED TO
LOOK AT THE QUESTIONS AND
PROVIDED ANSWERS TO THESE
QUESTIONS OFFLINE.
THANK YOU FOR ALL THE NIH STAFF
THAT WORKED IN THE BACKGROUND.
SO WHAT BOB AND I WOULD LIKE TO
DO IS TO NOW SUMMARIZE WHAT WE
THINK WE LEARNED FROM THE TWO
DAYS AND BOB, GENERATED A COUPLE
POWER POINT SLIDES WE WANT TO
BRING UP TO WRAP UP THE WORKSHOP
WHICH HAS TRULY BEEN TERRIFIC.
BOB, I'M GOING TO LET YOU GET
STARTED WITH THE SLIDES.
>>THANK YOU VERY MUCH.
I THINK I'LL NEED CAPACITY TO
SHARE SCREEN.
I WANT TO BEGIN BY THANKING THE
INCREDIBLE EFFORTS OF THE STAFF.
EVERYONE HAS BEEN PHENOMENAL IN
MAKING THIS GO FORWARD SO WELL.
RITA AND I REVIEWED HOW BEST TO
SUM UP AND RATHER THAN REPEATING
EVEN AT HIGH ALTITUDE WHAT'S
BEEN SAID ALREADY IN TERMS OF
THE DISCREET WORKING GROUPS AND
THEIR PRIORITIES, INSTEAD WE'D
SUMMARIZE THE COMMON THREADS OR
THEMES THAT HAVE BEEN INTERWOVEN
BETWEEN BOTH DAYS.
IT MAY BE USEFUL IN THINKING OF
THE TIGHTNESS OF THE DOCUMENT
AND THE BROAD CONSENSUS AS THE
BEEN FOR SOME OF THE CONCEPTS
AND IDEAS.
THE FIRST CATEGORY IS THE BASIC
AND CLINICAL RESEARCH IDEAS.
I THINK FOREMOST AMONG THESE AND
WE HEARD THIS FROM ALL THE
INVESTIGATORS AND CLINICIANS AND
THE NIH STAFF IS THE INCREDIBLE
URGENCY THERE IS TO TAKE THE
EXPANDING UNIVERSE OF NEW TOOLS
AND RESOURCES AND BRING THEM TO
BEAR POWERFULLY AND EFFECTIVELY
TO UNDERSTAND AND TREAT ALS THAT
UNDER LIES EVERYTHING WE
DISCUSSED.
THERE'S NO DOUBT A SECOND AND
CLEAR DOMINANT THEME WAS THE
CHALLENGE TO UNDERSTAND SPORADIC
ALS.
AS WE SAID, IN ALL THE MEETINGS
WHEN WE MEET BY ZOOM OR FACE TO
FACE, THE UNANSWERED QUESTION
THAT ALWAYS LOOMS IS WHAT IS
SPORADIC ALS.
WHILE IT'S TRUE GENETICS AND THE
LESSONS FROM UNDERSTANDING
FAMILIAL ALS HAVE PROVIDED SOME
INSIGHT INTO SPORADIC ALS WE CAN
DO BETTER AND MUST.
AND AMONG OTHER THINGS, AS
WALTER SAID AT THE START,
THERE'S AN IMPORTANT CHALLENGE
OF UNDERSTANDING INTERPLAY
BETWEEN GENETIC VARIANTS AND
ENVIRONMENT AND WHAT HE TERMED
THE DARK MATTER OF ENVIRONMENTAL
TOXINS.
I THINK THAT'S A THING THAT
SEEMS TO BE VERY CLEAR
THROUGHOUT ALL OF THIS.
A THIRD THEME THAT WE FREQUENTLY
HEARD AND POWERFULLY SO WAS
UNDERSTANDING HETEROGENEITY AND
SUB CATEGORIES OF EACH AND
IMPORTANTLY HOW THEY'RE RELATING
BETWEEN THE CLINICAL AND
MOLECULAR SUBSETS AND THE
INTERLOCKING TERMS.
ANOTHER IS THE IMPORTANCE OF
EARLY INTERVENTION AND
PRESYMPTOMATIC TREATMENT AND
THOSE WHO ARE CARRIERS EXAMPLE
CAN BE IDENTIFIED FOR EMERGING
THERAPIES.
WE HEARD ABOUT THE IMPORTANCE OF
COLLABORAT
COLLABORATIVE AND SHARED
INFRASTRUCTURE AND SHOULD BE
PROVISIONS FOR OPEN ACCESS.
THIS WAS LITERALLY A POINT MADE
IN EVERY WORK GROUP AND
DISCUSSED WELL THIS AFTERNOON
WITH THE DOCTOROFF GROUP WHO
STATED THE IMPORTANT POINT THAT
SYNERGIES THAT ARE DERIVED IN
THIS WAY WILL BASIC LEVERAGE AND
USE THE SHARED RESOURCES SOME
WHICH ALREADY EXIST AND SHOULD
INCLUDE MULTI-DIMENSIONAL DATA
SETS AND LONGITUDINALLY
CONNECTED SAMPLES AND IMAGING
AND HARMONIZED TO ALLOW THE
APPROPRIATE COMPARISON CONTRAST,
INTEGRATION AND DATA MINING TO
BE EMPLOYED.
AS WE THINK ABOUT INTERROGATING
THE DATA SETS WE ALSO LOOK AT
THE EMERGING TECHNOLOGIES IN
ARTIFICIAL INTELLIGENCE WHICH
MAY ENORMOUSLY ENHANCE OUR
ABILITY TO DISCERN MEANINGFUL
FINDINGS FROM THE SETS.
ALSO, MENTIONED WAS THE
IMPORTANCE OF DEVELOPING
REPOSITORIES FOR RESEARCH TOOLS,
PROTOCOLS AND RESOURCES AND
DEVICES WHICH WILL HELP ALL OF
US AND IMPORTANTLY THE DECREASED
BARRIERS FOR ENTRY INTO ALS
RESEARCH A POINT THAT LED TO OUR
LAST SHARED VIEWS AND IN
DIFFERENT PRESENTATIONS
EMPHASIZE EDUCATION IS IMPORTANT
AND WE HAVE TO THINK OF TRAINING
CLINICIANS, SCIENTISTS AND
GENETIC COUNSELORS AND OTHER WHO
JOIN THE ALS COMMUNITY TO
ENHANCE THE RESEARCH EFFORTS.
RITA.
>>THANK YOU, BOP.
THE SECOND BROAD TOPIC WE TRIED
TO SUMMARIZE THE HIGHLIGHT OF
THESE TWO DAYS AND THE
DISCUSSIONS WE HAVE WAS ON
PATIENT CARE AND ADVOCACY.
THAT'S BECOME OBVIOUS DURING OUR
SERVICE POSSESSION WHERE WE A
DECISION TO IMPROVE ON THE
QUALITY OF LIFE FOR PATIENTS AND
PEOPLE LIVING WITH ALS.
AND THE CHALLENGE IS WITH THE
QUALITY OF LIFE ISSUES IS TO
DEFINE AND DEVELOP THE FIRST
FUNDING METRICS AND THAT NEEDS
TO BE ADDRESSED.
AND WE MUST NOT LOSE SITE AND
THAT WAS AN IMPORTANT TOPIC OF
NOT JUST CAN WE PICK UP A CUP OF
COFFEE BUT ALSO THE WELLNESS AND
JOY OF PEOPLE LIVING WITH THIS
DISEASE DESPITE THE DISABILITIES
AND DYSFUNCTION THEY SHOW AND
PROGRESS DURING THE DISEASE.
IT WOULD BE IMPORTANT TO IMPROVE
ON THE RATING SYSTEM AND SCALES
UTILIZED IN THE CLINIC TO DATE
AND SHOULD THAT INCLUDE QUALITY
OF LIFE AND HOW WILL THAT BE
DEFINED AND HOW SHOULD WE
INTEGRATE THIS?
THE SECOND TOPIC IS A LITTLE BIT
OVERLAPPING.
AND WE SAW NOT ONLY DO WE HAVE
TO EDUCATE RESEARCHERS AND
CLINICIANS WE HAVE TO PROVIDE
ACCESS TO THE EDUCATION FOR PALS
AND CALS.
THERE SHOULDN'T BE LIMITATIONS
FOR ANYBODY BEING DIAGNOSED ALS
AND THEY GET ALL THE INFORMATION
THEY NEED TO UNDERSTAND THE
DISEASE AND WHAT'S COMING THEIR
WAY AND LEARN ABOUT RESOURCES
AVAILABLE TO THEM AND HOW TO GET
ACCESS AN EXAMPLE IS SOFTWARE TO
HELP THEM COMMUNICATE WHEN THEY
START LOSING THEIR ABILITY TO
SPEAK AND COMMUNICATE.
THERE NEEDS TO BE BETTER ACCESS
PLANS AND FOR OPPORTUNITIES FOR
PEOPLE LIVING WITH ALS AND
CAREGIVERS TO NOT HAVE TO GO
THROUGH HOOPS AND TAKE WEEKS AND
MONTHS TO FIGURE OUT HOW DO WE
ACCESS THE RESOURCES.
THAT SHOULD BE AN EASY ONE-SHOP
THAT PROVIDES ALL THE RESOURCES
AND ALLOW PEOPLE LIVING WITH ALS
AND FAMILY MEMBERS TO GET WHAT
RESOURCE THERE TO IMPROVE THEIR
RED QUALITY OF LIFE AND HAVE
ACCESS TO THEM AND GET THEM IN
THEIR HOMES.
AND WHAT CAME UP NOT JUST TODAY
BUT YESTERDAY FOR THE CLINICAL
TRIAL DESIGN AND INCLUSIVITY AND
IT'S AN IMPORTANT TOPIC WE
CANNOT IGNORE ANY LONGER AND WE
TALKED ABOUT THIS IN THE
DISCUSSION TODAY BUT IT'S
IMPORTANT TO INCLUDE A DIVERSITY
IN REGARD TO THE GENETICS AND
THE CLINICAL HETEROGENEITY OF
THE DISEASE AND WILL TEACH US
HOW HETEROGENOUS THE DISEASE IS
AS WE GO THE ROUTE OF BEING MORE
INCLUSIVE.
WE HAVE TO REACH OUT TO RURAL
LOCATIONS.
WE CANNOT JUST FOCUS OUR
RESEARCH ON THE PEOPLE WITH THE
DISEASE THAT LIVE CLOSE TO THE
CENTERS OF EXCELLENCE AND GLAD
WE HAD THE DISCUSSION KNE NEIL
MENTIONED AND DOESN'T MEAN WE'LL
EXCLUDE THOSE WHO DON'T HAVE THE
CENTERS OF EXCELLENCE.
IT'S IMPORTANT WE MAKE SURE THAT
WE'RE NOT EXCLUDEING ANYBODY NOT
LIVING IN BIG CITIES WHERE THE
CENTERS ARE OFTEN LOCALIZED AND
RACE AND GENDER WHICH CAN
INFLUENCE THE TIMELINESS OF
DIAGNOSIS AND THERAPIES AND HAVE
TO PAY ATTENTION TO THE ASPECTS
AND INCLUDE THIS IN THE STUDIES
WE'RE DOING AND INCLUDE THIS IN
THE STUDY DESCRIBE OF OUR
CLINICAL TRIALS AND IN OUR
RESEARCH STUDIES AND
PRE-CLINICAL AND TRANSLATIONAL
STUDIES WE'RE DOING.
THIS IS A HIGH PRIORITY CLEARLY
IDENTIFIED THROUGH THE
DISCUSSIONS WE HAD TODAY AND
YESTERDAY.
DO YOU WANT TO SAY ANYTHING
ABOUT THE THOUGHTS YOU HAD?
>>I WANT TO EMPHASIZE THAT IT'S
BEEN A WONDERFUL EXERCISE
PULLING THIS WHOLE DOCUMENT
TOGETHER WORKING WITH RITA AND
THE NIH STAFF TO PREPARE THE
DRAFT BUT IT'S STILL A DRAFT.
IT'S NOT TOO LATE TO LOOK AT IT
AGAIN AND ENCOURAGE THE
CO-CHAIRS AND ANY PARTICIPANT TO
LOOK AT WAYS IT COULD BE TWEAKED
AND TUNED AND IMPROVED.
IT WAS STRIKING, FOR EXAMPLE,
THAT IN A WONDERFUL SECTION ON
ACCELERATING BIOLOGY THERE WAS
MENTION FOR EXAMPLE OF GENETIC
RISK FACTORS BUT NOT A LOT ABOUT
COMBINATORIAL GENETIC AND WE
COULD TALK MORE IN THE DRAFT
ABOUT WHAT WE MEAN BY CHALLENGES
AND HAD A SUMMARY FROM WALTER
ABOUT THE NEW OFFICE OF
NEUROEXPOSE AND TOXICOLOGY AND
THE THERAPEUTIC PIPELINE.
MY POINT IS WE WANT IT HEAR FROM
YOU.
WE WANT TO KNOW IF THERE ARE
WAYS I THINK WITHOUT MAJOR
STRUCTURAL REDESIGN WE CAN
SHARPEN THE DOCUMENT BECAUSE
RIGHT NOW IT REFLECT THE
REFLECTION FROM BEING HIGH
ALTITUDE AND BE USEFUL IN
GUIDING THE NINDS IN TERMS OF
THE STRATEGIC PRIORITIES.
AGAIN, AT THIS POINT I WANT TO
THANK EVERYONE AND WHAT A
PRIVILEGE IT'S BEEN INVOLVED AS
A CO-CHAIR AND AS THE WORK YET
TO BE DONE BUT WE'RE ON THE WAY.
AT THIS POINT WE TURN TO WALTER
FOR ADDITIONAL COMMENTS.
>>WHAT A GREAT TWO DAYS.
I THINK WE HEARD ABOUT THE
TREMENDOUS ENERGY THERE IS IN
THIS COMMUNITY TO TRY TO MAKE A
REAL DIFFERENCE FOR PEOPLE WHO
HAVE ALS PEOPLE AT RISK FOR ALS
AND THOSE WHO GET IT IN THE
FUTURE AND DON'T HAVE ANY IDEA
YET THIS IS GOING TO HAPPEN TO
THEM.
FROM THE NINDS STANDPOINT, WE
ARE STICKING WITH THIS PLAN.
WE'RE NOT GOING AWAY.
WE THINK THIS IS INCREDIBLY
IMPORTANT YOU DIDN'T HAVE TO
CONVINCE US.
WE'VE KNOWN IT'S IMPORTANT AND
ALWAYS FELT BAD WE DIDN'T HAVE
THE THERAPIES WE WANT.
TO GET THE INPUT WILL HELP US.
AND THERE'S IDEAS IN A BIT BUT I
DID WANT TO GO THROUGH SOME OF
THE IMPORTANT NEXT STEPS AND WE
DID A LOT OF HEAVY LIFTING ON
OUR END AND OUR PROGRAM DIRECTOR
FOR ALS FOR A LONG TIME.
I WANT TO THANK EVERYONE FOR
PARTICIPATING WE HAD OVER 400
PEOPLE REGISTER FOR THE MEETING.
WE HAD TREMENDOUS INVOLVEMENT
RIGHT THROUGH.
WE HAD THE PUBLIC COMMENTS WERE
INCREDIBLY VALUABLE AND GAVE US
A LOT TO THINK ABOUT AS WE TRY
TO TACKLE THIS DIFFICULT AND
IMPORTANT PROBLEM.
I WANT TO GIVE A BIG THANKS TO
THE STEERING COMMITTEE AND
WORKING GROUPS WHO WORKED MANY
MONTHS.
THIS IS GOING ON FOR A YEAR NOW
ON DEVELOPING THE PRIORITIES.
AND THE NINDS STAFF AND EMILY
WHO RUNS THE SMALL BUSINESS
PROGRAM AND IT WILL BE CRITICAL
IN DEVELOPING THESE WITH
INDUSTRY AND SAM WHITE AND
REBECCA AND CHRISTINE WHO SPENT
SO MUCH TIME AND MOST
IMPORTANTLY TO THOSE WHO
PARTICIPATED AND SHARED YOUR
THOUGHTS AND IDEAS.
AND THE GROUP WILL TAKE THE
INPUT THEY RECEIVED HERE AND THE
COMMENTS COMING IN FROM THE
PUBLIC COMMENT PERIOD AND REVISE
THE DRAFT PRIORITIES WE'LL POST
THE REVISED DOCUMENT FOR FOR AT
LEAST 30 DAYS AND THE FINAL
PRIORITIES WILL THEN BE
PRESENTED TO THE COUNCIL
FEBRUARY 1 AND 2.
THE COUNCIL, I'M SURE, HAVING
SEEN THE QUALITY OF THE DOCUMENT
AND SURE THE COUNCIL OF OUR
INSTITUTE WILL HAVE SUGGESTIONS
AND COMMENTS AND IN TERMS OF
IMPLEMENTATION, WE'VE DONE THIS
BEFORE, THIS IS SOMETHING WE'VE
DONE IN OTHER DISEASES.
I'LL TALK ABOUT A COUPLE THINGS
THAT HAVE HAPPENED BEING
PREPARED HAS SOMETIMES TURNED
OUT TO BE INCREDIBLY HELPFUL.
SO JUST TWO EXAMPLES OVER A
DECADE AGO WE WERE ASKED TO
DEVELOP A PLAN FOR ALZHEIMER'S
RESEARCH.
A PROCESS HAPPENED MUCH LIKE
THIS AND WHAT FOLLOWED WAS
CONGRESS ASKED FOR A BUDGET TO
PUT THAT PLAN TOGETHER.
THEY ASKED FOR THE BUDGET TO
BYPASS HHS AND GO STRAIGHT TO
CONGRESS AND ACTUALLY FUNDED
THAT PLAN.
WHEN PEOPLE ARE WORKING ON IT
THERE WAS A LOT OF CYNICISM BUT
NO MORE CYNICISM.
IT ALL CAME TO FRUITION.
AND WE WORKED ON A PLAN FOR A
FEDERAL PAIN RESEARCH STRATEGY
ACROSS THE COUNTRY.
WIZ WAS PUBLISHED.
THERE WERE IMPROVEMENTS MADE BUT
THEN THE HEAL INITIATIVE CAME
AND CONGRESS APPROPRIATED
SIGNIFICANT AMOUNTS OF FUNDS AND
WE HAD CONSIDERABLE FUNDING.
THERE'S NOTHING BEING PREPARED
AND WITH THIS GROUP WE PREPARED.
NOW WE HAVE TO USE THE RESOURCES
WE PUT TOGETHER TO MAKE A
DIFFERENCE.
AND TO DRIVE THE ENABLERS AND
GIVE THEM THE RESOURCES THEY
NEED TO MAKE THE BIGGEST
DIFFERENCE.
YOU KNOW THERE'S GOING TO BE
DISCOVERIES AND ADVANCES BUT
IT'S HARD TO PREDICT WHERE
THEY'LL COME FROM.
WE HAVE TO BE CAREFUL ABOUT
GOING DOWN A TUNNEL WHEN THE
ADVANCES CAN COME MANY MILES
FROM WHERE YOUR DIGGING.
YOU HAVE TO BE MORE STRATEGIC IN
TERMS OF ENABLING LOTS OF
FLOWERS TO BLOOM WHERE THERE'S A
SINGULAR FOCUS AND HERE IN ALS,
IT'S CLEAR WHAT THE FOCUS IS
BETTER TREATMENT NOR DISEASE AND
WHAT IT TAKES TO GET TO THE
TREATMENT AND MOVE IN THAT
DIRECTION.
THAT WOULD BE OUR PLAN.
I WON'T BE ABLE TO TALK ABOUT
ALL THE THINGS THAT CAME UP
TODAY BUT I WOULD SAY A COUPLE
THINGS AND THERE IS A
MISPERCEPTION NIH MAKES THINGS
HAPPEN.
IT'S A GRANTING AGENCY.
FOR THINGS TO HAPPEN, PEOPLE
HAVE TO COME TOGETHER LIKE WE
CAME TOGETHER HERE AND DEVOTE
THEMSELVES TO A PROJECT AND
SUBMIT IT AS A GRANT AND THAT'S
HOW THE MONEY GOES OUT TO GET
THINGS DONE.
THA
THAT'S LEGISLATIVE.
THERE'S NO EXCEPTIONS EXCEPT FOR
LIKE COVID.
THAT'S THE PROCESS THAT'S MADE
NIH WHAT IT IS.
RIGHT NOW WITH THIS PLAN, AS HAS
BEEN SEEN, IT WON'T BE TURNED
UPSIDE-DOWN AND PEOPLE CAN
PEOPLE CAN START TO GALVANIZE ON
WHAT'S IMPORTANT TO DO AND WE
KNOW WHERE WE WANT TO GO IN THE
END BUT I WON'T TELL PEOPLE NOT
TO DO THING IF IT FITS.
AND IT DEPS ON WHAT THE
INVESTIGATORS AND PATIENTS WHO
WORK WITH THE INVESTIGATORS ON
THE OUTSIDE REALLY WANT TO DO
AND THERE'S NOTHING TO STOP THAT
FROM HAPPENING LIKE TOMORROW.
AND IF YOU DON'T HAVE A
MECHANISM FOR EVERY SINGLE
DISEASE YOU'D BE PARALYZED.
INSTEAD WHAT WE'VE DONE OVER THE
YEARS IS DEVELOP MECHANISMS THAT
WOULD ENABLE PRETTY MUCH
ANYTHING TO HAPPEN TO MOVE A
DISEASE FORWARD.
MANY THINGS WE TALKED ABOUT, ARE
DOABLE IMMEDIATELY.
AND WE HAVE BIOMARKERS.
WE HAVE BIOMARKER ANALYTIC
VALIDATION AND CLINICAL
VALIDATION AND HAVE BIOMARKERS
IN PHASE 2 TRIALS.
WE HAVE NETWORKS SET UP TO DO
PHASE 2 TRIALS.
AND THE KEY THING FOR NINDS IS
TO BRING THESE TOGETHER AND JUST
LETTING THE FLOWERS BLOOM CAN
GET YOU INEFFICIENCIES AS WELL
AS INNOVATION.
AND INEFFICIENCY IS NOT
ACCEPTABLE GIVEN THE URGENCY OF
THE DISEASE AND DEVELOPING AN
ORGANIZATION DRIVING ALS
RESEARCH FORWARD SO THE ULTIMATE
SHARED GOAL WE ALL HAVE IS WHAT
WE CAN DO TOGETHER AS A COMMUNED
AND THE GOVERNMENT I THINK WE
DON'T WANT TO TAKE OVER THE
WORLD BUT WE THINK WE CAN MAKE A
BIG DIFFERENCE IN THAT SPACE.
REALLY LOOKING FORWARD TO THE
PUBLIC-PRIVATE PARTNERSHIP WITH
THE FDA AND ANOTHER SPACE WHERE
THE WORK DONE IN THE LAST TWO
DAYS DOESN'T END AND GOES ON
UNTIL WE GET TO THE FINAL CALL.
AND WE BROUGHT IN DATA FROM
DIFFERENT STREAMS AND WE HAVE
TRANSCRIPTOMIC DATA AND CLINICAL
DATA AND WHOLE GENOME SEQUENCING
DATA AND HAVE ABOUT 10,000
PEOPLE IN THAT DATABASE RIGHT
NOW AND IT'S OPEN SOURCE AND
ANYBODY CAN GO IN AND MINE THE
DATA AND THE PORTAL IS A
SOPHISTICATED PORTAL AND THAT
CAN BE GALVANIZING AND CAN BE AN
ORGANIZING PRINCIPLE WHERE THE
DATA COMES IN AND THE PEOPLE WHO
ARE CONTRIBUTING ARE SEEING THE
VALUE OF BRINGING THAT DATA INTO
A REAL ORGANIZED PORTAL AND CAN
GALVANIZE A LOT OF BECAUSE YOU
DEVELOP STANDARDS ONES THEY'RE
DEVELOPED, EVERYBODY WILL USE
THEM AND THAT MAKES THE DATA
MORE COMPARABLE AND YOU CAN JUST
ADD ON WITH WHAT'S IN THERE
UNTIL PEOPLE CAN MAKE THE
DISCOVERIES THEY WANT TO MAKE.
AND THAT'S THE INDUSTRY AS WELL
AS ACADEMIC PEOPLE AND THE
PEOPLE LOOKING AT DATA ARE NOT
THE DOCTORS.
AT MEDICAL SCHOOL WE WERE NOT
TRAINED IN BIG DATA ANALYTICS.
SO WE NEED PEOPLE FROM THE TECH
FIELD TO HELP GET THAT GOING
WHEN IT COMES IN.
AND CPAP HAS A RECORD FROM
BRINGING IN DATA FROM COMPANIES
AND PLACEBO DATA AND TRIALS
ALONG WITH ACADEMICS AND WE HAVE
PEOPLE WHO ARE HIGH-LEVEL
ANALYTICS FOLKS TO LOOK AT THE
DATA.
THAT'S ONE POSSIBILITY AS WELL.
AND THE AMP IS THE INDUSTRY
ADVOCACY ORGANIZATION AND NINDS
AND NIH PARTNERSHIP.
AND I THINK THOSE THINGS ARE
DOABLE.
THEY'LL TAKE TIME TO SET UP AND
I THINK THE AMP PORTAL TOOK US
MAYBE THREE YEARS TO SET UP.
IT'S AN INCREDIBLE AMOUNT OF
WORK TO TRANSFORM THE DATA
COMING IN HAVE VARIOUS SOURCES.
IT'S COMPARABLE DATA.
THE COST IS NOT INSIGNIFICANT.
IT WAS ABOUT $25 MILLION FOR THE
PARKINSON'S AMP AND IT TAKES
PEOPLE WILLING TO DO THAT.
AND LOOKING AT THE BIOMARKER
PROGRAMS AND PARKINSON'S.
THE PEOPLE WHO DID THEY'LL WORK
AND BROUGHT IN THE DATA
TOGETHER.
WE CAN'T FORCE THAT TO HAPPEN.
WE CAN CERTAINLY INCENTIVIZE IT.
THE FDA ANNOUNCED THEY'LL BE
LAUNCHING IN THEIR
NEURODEGENERATIVE DISEASE
NATURAL HISTORY PROGRAM AND ALS
NATURAL HISTORY TRIAL AT THE
UNIVERSITY OF MINNESOTA AND WE
WILL BE WORKING WITH THEM ON
THAT PROCESS.
AND WITH THE DATA, AFTER MANY
YEARS, FINALLY NIH HAS MOVED TO
A DATA POLICY PRETTY STRINGENT.
AS OF JANUARY, ANY GRANT COMING
IN THE DATA HAS TO BE POSTED
THAT'S GOING CHANGE OUR SYSTEM.
WHAT NIH HAS NOT DONE IS
MANDATED THE DATA IS POSTED IN A
PARTICULAR FASHION AND THAT
THEY'RE LEAVING UP TO THE
SCIENCE COMMUNITY.
THERE ARE GRANT APPLICATIONS OUT
NOW SO BRING DATA IN AND -- TO
BRING DATA IN AND THE COMMUNITY
CAN DECIDE WHAT THE DATA SHOULD
LOOK LIKE AND START NOW IN TERMS
OF BRINGING THE DATA IN.
ANOTHER EXAMPLE OF MECHANISMS
OUT THERE TO DO A LOT OF THE
THINGS THAT PEOPLE WANT TO DO
AND WE ARE CERTAINLY GOING TO
PUT PLANS TOGETHER AND THERE'LL
BE PROGRAMS THAT FOLLOW THIS
PLAN BUT IN TERMS OF THE TIME
FRAME IF YOU THINK IT'S URGENT,
THEN DON'T WAIT.
LOOK AT WHAT'S AVAILABLE AND
CALL ME, WHATEVER YOU WANT TO
DO, I THINK WE CAN FIND OUT A
WAY TO DO WITHOUT WAITING AND
THINGS WILL GET BETTER AND
BETTER BECAUSE OF THE WORK YOU
DO FOR PEOPLE WITH ALS AND THE
WORK THE PEOPLE AT ALS DO TO
HELP SOMETIMES THEY'RE DOING
THIS TO HELP OTHERS AND PUTTING
OUT MONEY TO GO TO SAN FRANCISCO
THAT'S ALTRUISM MORE THAN ANYONE
SHOULD EXPECT ANYBODY TO DO.
WE HAVE TO DO THE BEST WE CAN TO
TRY TO THE QUALITY OF LIFE OF
PEOPLE LIVING WITH THE DISEASE
IS A STRONG STATEMENT THAT CAME
OUT.
THE FOCUS AS I SAID IS TO GET A
TREATMENT SO PEOPLE DON'T SUFFER
BUT WE ALSO HAVE TO THINK ABOUT
THINGS WE CAN DO AND THERE ARE
THINGS WE CAN DO TO ADVANCE THE
QUALITY OF LIFE FOR PEOPLE WHO
HAVE THE ILLNESS.
AND SO I THINK THAT WAS ANOTHER
REALLY STRONG SENTIMENT OF THE
GROUP.
WITH THAT ONCE AGAIN, THINK
ABOUT WHAT YOU HEARD THINK ABOUT
WHAT YOU WANT TO DO AS A
COMMUNITY AND LET'S GET STARTED.
THEN WE'LL BE WORKING WITH THIS
PLAN TO REALLY TRY TO PUT A LOT
MORE MUSCLE INTO THE WORK YOU
WANT TO GET DONE FOR PEOPLE WITH
ALS.
WITH THAT, THANKS SO MUCH AND
THANKS TO BOB AND RITA AND I
HATE TO HAVE THE LAST WORD SO
RITA, HAVE THE WORD.
>>THANK YOU, WALTER.
>>MY LAST WORD IS TO THANK ALL
OF THE NIH STAFF.
IT WAS REALLY THEM WHO PULLED US
TOGETHER WHO REMINDED US TO GIVE
US THE DEADLINE WHO PROVIDED A
FRAMEWORK THAT ALLOWED ALL THE
WORKING GROUP MEMBERS TO REALLY
ACCOMPLISH WHAT THEY HAVE
ACCOMPLISHED.
IT WAS AN INCREDIBLE
ACCOMPLISHMENT AND I THINK
EVERYONE CAN PAT THEMSELVES ON
THE SHOULDER.
BOB WAS INCREDIBLE TO WORK WITH.
IT WAS A PLEASURE TO DO THIS
WITH HIM AS A CO-CHAIR AND I
COULD NOT HAVE ASKED FOR A
BETTER PERSON TO LEAD THE
EFFORT.
IT'S BEEN WONDERFUL TO WORK WITH
EVERYBODY AND WE LOOK FORWARD TO
CONTINUE THE WORK, INTEGRATE THE
COMMENTS FROM THE AUDIENCE TODAY
AND YESTERDAY TO MAKE IT A
BETTER STRATEGIC PLAN WE PUT
TOGETHER THAT WE THEN PRESENT TO
COUNCIL AND HOPEFULLY FROM THERE
GREAT THINGS WILL HAPPEN WITH
WHAT WE ACCOMPLISHED.
THANK YOU, EVERYBODY.
>>I WOULD SAY, I STARTED
LEARNING FROM BOB IN 1981 AND
I'M STILL LEARNING.
>>THANK YOU, ALL.
GREAT DAY.
>>THANK YOU.
, EVERYBODY.
>>THANK YOU.