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
MJFF IS THE WORLD’S LARGEST NONPROFIT FUNDER OF PD RESEARCH 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 » $108 million in research programs by actor Michael J. Fox funded including education, policy work » Since inception, 88 cents of every and other program activities in 2018 » More than 700 active grants in current dollar spent goes to research programs portfolio » More than $900 million in research » 32% of funded projects led by programs funded to date ( 3,000+ researchers projects) outside the United States In 2019, we hope to put another $100 million to work across our research programs. 3
WE KNOW SCIENCE… AND THE BUSINESS OF SCIENCE 39 1 12 11 Our team of experts identifies and manages the most compelling Parkinson’s science and applies business principles to speed progress. 4
PATIENTS’ NEEDS DRIVE OUR EFFORTS: WE URGENTLY DEPLOY DONATIONS TO THE MOST PROMISING PROJECTS The Michael J. Fox Foundation has no endowment and 88¢ of every dollar spent goes directly to research. 5
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 6
PARKINSON’S RESEARCH UPDATE
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. 8
HOW CAN WE SLOW, STOP, OR REVERSE THE PROGRESSION OF PD? 9
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. 10
WE’RE SUPPORTING VARIED APPROACHES TO STOP THE DISEASE PROCESS 11
ALPHA SYNUCLEIN PROGRAMS THAT HAVE TRANSITIONED TO THE CLINIC
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 13
GENETIC DISCOVERIES CAN POINT TO WIDESPREAD TREATMENTS Develop treatments Link the gene • How can we to biology Find the gene safely target the protein? • How do mutations • Look for families • Another place to impact protein with PD target and offset function? • Examine large dysfunction? • How does altered populations of • Will therapy help function link to PD? people with and wider PD • Do people without without PD population? mutation have same dysfunction?
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). Genetic Phase I Phase II Phase III Targets GBA1 LRRK2 Scientists are also working on therapies against mutations in the PRKN and PINK1 genes. 15
BETTER TREATMENTS FOR THE MOTOR AND NON-MOTOR SYMPTOMS OF PD 16
STUDIES TO BETTER UNDERSTAND AND TREAT SYMPTOMS Sleep Problems Depression and Anxiety Memory Changes Vision Disturbances Speech and Swallowing Smell Loss Problems Low Blood Fatigue Pressure Pain Gait Urinary Problems and and Constipation Balance
DEVELOPMENTS IN CARE FOR PD SYMPTOMS In the past 6 years, 12 new Parkinson’s therapies have been approved and counting… Motor Symptoms Non-Motor Symptoms APPROVED APPROVED » Levodopa/Carbidopa : Duopa (2015) & Rytary (2015) » Low Blood Pressure: Northera (2014) » Dyskinesia : Gocovri (2017) » Psychosis: Nuplazid (2016) » ‘Off’ Periods : Xadago (2017) » Drooling : Xeomin (2017) » Extended-Release Amantadine : Osmolex ER (2018) IN-DEVELOPMENT » DBS Improvements: Infinity DBS system (2016) & » Constipation Vercise DBS system (2017) » Depression & Anxiety » Tremor (not controlled with medication) : Focused ultrasound (2018) » Cognitive Impairment » Rescue Therapies : Inbrija (2019) » Sleep Disorders IN-DEVELOPMENT » Rescue Therapies: Apomorphine strip (FDA) 18
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. 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. HWA Device Vibrating socks WOLK active airbag (Honda T&D. Ltd., Wako, Japan) prototype system 19
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. 20
FIELD-WIDE CHALLENGES 21
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 Researchers have strong leads for Parkinson’s biomarkers. 22
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)
SCIENTISTS ARE LOOKING FOR WAYS TO MEASURE PARKINSON’S Objective measures of disease – biomarkers – could diagnose and track Parkinson’s and speed clinical trials . Imaging changes in the living brain Measuring differences in body fluids and tissues Monitoring disease with wearable devices and other technologies
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 Learning more about the disease can point to new treatment approaches and allow faster testing of potential therapies.
WHAT CAN YOU DO TODAY
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