THE MICHAEL J. FOX FOUNDATION FOR PARKINSON’S RESEARCH

Mary McQuillenAssociate Director, Advancement

OUR CHALLENGES DON’T DEFINE US.OUR ACTIONS DO.

- MICHAEL J. FOX

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Our Mission

The Michael J. Fox Foundation is dedicated to finding a cure for Parkinson’s

disease through an aggressively funded research agenda and to ensuring the

development of improved therapies for those living with Parkinson’s today. Key Stats

» Public charity founded in 2000

by actor Michael J. Fox

» Since inception, 88 cents of every

dollar spent goes to research

programs

» More than $900 million in research

programs funded to date (3,000+

projects)

» $108 million in research programs

funded including education, policy work

and other program activities in 2018

» More than 700 active grants in current

portfolio

» 32% of funded projects led by

researchers

outside the United States

MJFF IS THE WORLD’S LARGEST NONPROFIT

FUNDER OF PD RESEARCH

In 2019, we hope to put another $100 million to work across our research programs.

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Our team of experts identifies and manages the most compelling

Parkinson’s science and applies business principles to speed progress.

WE KNOW SCIENCE…

AND THE BUSINESS OF SCIENCE

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The Michael J. Fox Foundation has no endowment and

88¢ of every dollar spent goes directly to research.

PATIENTS’ NEEDS DRIVE OUR EFFORTS:

WE URGENTLY DEPLOY DONATIONS TO

THE MOST PROMISING PROJECTS

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MJFF PLAYS A VITAL ROLE

IN DRIVING DRUG DEVELOPMENT

MJFF has focused on moving projects through the pipeline to

ultimately “de-risk” the PD field for commercial investors.

➢ Invest in riskier projects (early stage, minimal preliminary data)

➢ Encourage unprecedented collaboration between competitors in the

industry

PARKINSON’S RESEARCH UPDATE

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We’re making strides across priorities

We prioritize and pursue research in these areas, providing financial and intellectual

support for projects to ensure progress toward new therapies for people with PD.

HOW CAN WE SLOW, STOP, OR REVERSE THE PROGRESSION OF PD?

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THERAPIES TO SLOW OR STOP PARKINSON’S

» Alpha-synuclein

– Target protein that clumps in brain cells of everyone with PD; one of our most

promising routes.

– Ten therapies across Phase I and II that include a vaccine and antibodies against

alpha- synuclein as well as a pill that could break up clumps

» GBA and LRRK2

– Most common genetic links to Parkinson’s are mutations in these genes.

– Three therapies against GBA are in human trials, and scientists are investigating if

those treatments may benefit patients who do not carry the mutation.

– First drug trial of LRRK2 launched in 2017 and two more companies are expected to

begin trials in 2019.

» Other Therapies

– Initiatives testing therapies repurposed from other diseases.

– Studies learning more about cellular dysfunction to develop new drugs.

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WE’RE SUPPORTING VARIED APPROACHES

TO STOP THE DISEASE PROCESS

ALPHA SYNUCLEIN PROGRAMS THAT

HAVE TRANSITIONED TO THE CLINIC

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GENETICSAbout 10 percent of Parkinson's cases have been linked to a

genetic correlation; most cases are idiopathic.

» Millions of genetic differences make us who we are: from eye color to

risk for disease

» So far scientists have linked ~80 genetic regions to Parkinson’s disease

– Ten years ago, we knew only about ~10

» Some genes linked to Parkinson’s are pointing to ways we may stop the

disease: SNCA, LRRK2, GBA1

» Cells use the recipes in our genes to make proteins, which do many jobs

such as carry oxygen or digestion

» Rare variations in a gene are called mutations. These mutations cause

protein dysfunction, which can play a role in disease

GENETIC DISCOVERIES CAN POINT

TO WIDESPREAD TREATMENTS

Find the gene

• Look for families with PD

• Examine large populations of people with and without PD

Link the gene to biology

• How do mutations impact protein function?

• How does altered function link to PD?

• Do people without mutation have same dysfunction?

Develop treatments

• How can we safely target the protein?

• Another place to target and offset dysfunction?

• Will therapy help wider PD population?

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Scientists are also working on therapies against mutations

in the PRKN and PINK1 genes.

HOW DO WE GET TO A CURE?

Finding genetic differences in people with Parkinson’s can point to

precision therapy approaches (much like in cancer treatment).

Phase I Phase II Phase III

GBA1

LRRK2

Genetic

Targets

BETTER TREATMENTS FOR THE MOTOR AND NON-MOTOR SYMPTOMS OF PD

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Low Blood

Pressure

Speech and

Swallowing

Problems

Urinary Problems

and Constipation

Smell Loss

STUDIES TO BETTER UNDERSTAND AND

TREAT SYMPTOMSSleep Problems Depression

and Anxiety

Vision Disturbances

Fatigue

Pain

Gait

and

Balance

Memory Changes

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Motor SymptomsAPPROVED

» Levodopa/Carbidopa: Duopa (2015) & Rytary (2015)

» Dyskinesia: Gocovri (2017)

» ‘Off’ Periods: Xadago (2017)

» Extended-Release Amantadine: Osmolex ER (2018)

» DBS Improvements: Infinity DBS system (2016) &

Vercise DBS system (2017)

» Tremor (not controlled with medication): Focused

ultrasound (2018)

» Rescue Therapies: Inbrija (2019)

IN-DEVELOPMENT

» Rescue Therapies: Apomorphine strip (FDA)

DEVELOPMENTS IN CARE FOR PD SYMPTOMS

Non-Motor SymptomsAPPROVED

» Low Blood Pressure: Northera (2014)

» Psychosis: Nuplazid (2016)

» Drooling: Xeomin (2017)

IN-DEVELOPMENT

» Constipation

» Depression & Anxiety

» Cognitive Impairment

» Sleep Disorders

In the past 6 years, 12 new Parkinson’s therapies have been approved

and counting…

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Gait & Balance» MJFF is currently supporting eight projects testing assistive devices and rehabilitation programs

to prevent and ease gait and balance problems which can lead to falls and significant injury.

APPLIED TECHNOLOGIES FOR ADDRESSING

PD SYMPTOMSCurrent medications don’t address all non-motor symptoms. Scientists are

looking at medications and non-pharmacological interventions to treat other

troubling Parkinson’s symptoms.

Vibrating socks

prototypeWOLK active airbag

system

HWA Device

(Honda T&D. Ltd., Wako, Japan)

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WHAT ELSE MAY HELP MANAGE

PARKINSON’S SYMPTOMS?» Exercise

– No “right” exercise for Parkinson’s disease.

– Some (yoga, tai chi, boxing) may help with balance and flexibility.

– Work with a physical therapist to build a safe regimen you enjoy.

» Other Activities

– Meditation can relieve stress.

– Getting involved in your community or with favorite hobbies or creative projects

(dance, art) can keep you engaged and avoid isolation.

» Diet

– Healthy, balanced diet with antioxidants can promote general wellness.

– Some adjustments may help with medication effect and with non-motor symptoms.

– Talk to your doctor before adding any supplements to your treatment regimen.

FIELD-WIDECHALLENGES

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Researchers have strong leads for Parkinson’s biomarkers.

WHAT WILL HELP US MOVE FASTER?

Biomarkers: biological characteristics that alert us to risk, onset

or progression of disease

There are no validated biomarkers

of Parkinson’s yet.

These tools would help with:

» Earlier diagnosis and therapeutic intervention

» Charting disease progression

» Designing clinical trials

» Testing the impact of new therapies

LANDMARK STUDY TO DEFINE

PARKINSON’S AND PD PROGRESSION

MJFF launched the

Parkinson’s Progression Markers Initiative

in 2010 to validate biomarkers of PD

»Natural history study collecting clinical data, imaging, biosamples over time

» Following participants 5+ years

» Taking place at 33 clinical sites around the world

» Three cohorts:

» People with newly diagnosed PD and control volunteers

» People with early symptoms associated with PD risk (smell loss & RBD)

» People with & without PD with genetic mutations associated with PD

(LRRK2, GBA, and SNCA)

Objective measures of disease – biomarkers – could

diagnose and track Parkinson’s and speed clinical trials.

SCIENTISTS ARE LOOKING FOR WAYS

TO MEASURE PARKINSON’S

Imaging changes

in the living brain

Monitoring disease

with wearable devices

and other technologies

Measuring

differences

in body fluids

and tissues

Learning more about the disease can point to new treatment approaches

and allow faster testing of potential therapies.

GROWING UNDERSTANDING OF PD

Scientists are learning more about what goes wrong in Parkinson’s

and drawing lines between observations:

➢Naming more genetic risk factors (LRRK2, GBA)

➢Seeing changes in brain scans (dopamine activity, free water in

cells)

➢Linking genetic/clinical factors to symptoms (cognitive impairment,

psychosis)

➢Using new technologies (breath tests, WiFi, wearable devices) to

possibly diagnose PD

WHAT CAN YOU DO TODAY

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HOW CAN WE SPEED RESEARCH?

The faster volunteers enroll in studies, the sooner we will have

new treatments, new insights and, ultimately, a cure.

▪ Clinical trials need more participants. On the whole, the need for volunteers is

bigger than the pool of volunteers ready to participate in clinical trials.

▪ Many are unfamiliar with the research process and the ways to get involved.

▪ Better understanding of the PD community’s perspectives on clinical research

helps trial teams identify research priorities and address gaps in study

enrollment.

THOUGH 88%of people with Parkinson’s

say they would be willing

to participate in a trial

FEWER Than 25%ever do

Fox Trial Finder:

▪ Connects volunteers with recruiting trials

▪ Allows direct communication between you and trial teams

Fox Insight:

▪ An online clinical study that empowers patients, control

volunteers and researchers to work together toward

Parkinson’s breakthroughs.

▪ Over 30,000 participants to date

Donate:

▪ To make an immediate impact, gifts of cash can be made

online or by check. We also welcome gifts of stock or IRA

distributions.

▪ Join the Legacy Circle by naming MJFF in your estate

plans or by establishing a Charitable Gift Annuity.

YOU ARE NEEDED TO SPEED RESEARCH

foxtrialfinder.michaeljfox.org

foxinsight.michaeljfox.org

michaeljfox.org/donate

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Direct-from-patient information provides researchers a unique and critical

lens for understanding the impact of disease and treatment.

Through quarterly study visits,

participants fill out a series of

questionnaires to provide critical

insights on:

▪ Symptoms

▪ Physical function

▪ Medications

▪ Quality of life

▪ Unmet Needs

▪ Healthcare preferences

YOUR EXPERIENCE FUELING RESEARCH

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Policy efforts are central to our research

mission

Further Parkinson’s

Disease Research

Support Drug Development

and Approvals

Safeguard Access

to Care and Support

As more promising PD drug candidates reach late-stage testing and enter complex

regulatory and reimbursement approval processes, MJFF advocates for programs

that accelerate therapeutic development and reduce barriers to care and support

services.

The Foundation focuses on three policy priority areas:

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RESOURCES

STAY INFORMED AND UP TO DATEVisit www.michaeljfox.org for the latest

on Parkinson’s research and care.

Webinars: Live panel discussions each month

and available anytime on-demand (Free CME credit available)

www.michaeljfox.org/webinars

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Ask the MD: A movement disorder specialist

tackles Parkinson’s topics through videos and blogs

www.michaeljfox.org/ask-the-md

Podcasts: Expert interviews on life with Parkinson’s

and the latest science

www.michaeljfox.org/podcasts

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Topics covered:

▪ Basics of clinical research

▪ Need for diversity in trials

▪ Importance of genetic research

NAVIGATING CLINICAL TRIALS: A GUIDE FOR

PATIENTS AND FAMILIESA comprehensive guide that demystifies the research process, as well as

stories and videos featuring people with Parkinson's and their loved ones.

Visit

www.michaeljfox.org/ParticipantPack

to download the guide and watch

accompanying video.

THANK YOU!

QUESTIONS?