THE MICHAEL J. FOX FOUNDATION FOR PARKINSONS RESEARCH Mary - - PowerPoint PPT Presentation

THE MICHAEL J. FOX FOUNDATION FOR PARKINSON’S RESEARCH

Mary McQuillen Associate 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

• utside 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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GENETICS

About 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 SYMPTOMS

Sleep Problems Depression and Anxiety Vision Disturbances Fatigue Pain Gait and Balance Memory Changes

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Motor Symptoms

APPROVED

» 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 Symptoms

APPROVED » 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 SYMPTOMS

Current 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 prototype WOLK 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-WIDE CHALLENGES

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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

• r progression of disease

There are no validated biomarkers

• f 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%

• f 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

• nline 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 DATE

Visit www.michaeljfox.org for the latest

• n 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 FAMILIES

A 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?