STUDIES TO SLOW OR STOP PARKINSON S DISEASE. MJFF Third Thursdays Webinar October 15, 2015

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1 STUDIES TO SLOW OR STOP PARKINSON S DISEASE MJFF Third Thursdays Webinar October 15, 2015

2 WHAT WE LL COVER TODAY What are the varied approaches to stop Parkinson s? How can our immune system help fight Parkinson s disease? What have we learned from observing people with PD? What happened to neurotrophic factors? Why is it so difficult to test a therapy that modifies the disease process? 2

3 OUR PANELISTS Moderator: Dave Iverson Founding Member, MJFF Patient Council Panelists: Bill Shepherd Chief Financial Officer, Global Wealth and Investment Management, Bank of America MJFF Leadership Council Member Lorraine V. Kalia, MD, PhD, FRCPC Neurologist, Toronto Western Hospital Assistant Professor, University of Toronto Todd Sherer, PhD CEO, The Michael J. Fox Foundation 3

4 HOW ARE WE TRYING TO STOP PARKINSON S? oprevent, break up or clear out toxic clumps of the protein alpha-synuclein olearn from other studies what compounds are associated with lower risk of PD and/or slower progression ointroduce therapies that protect brain cells There are many therapies with the potential to slow or stop PD already in human testing. 4

5 HOW CAN OUR IMMUNE SYSTEM HELP FIGHT PARKINSON S? Antibodies are Y-shaped proteins that identify and neutralize harmful viruses and bacteria. Parkinson s researchers are using antibodies to neutralize clumps of the protein alpha-synuclein, which clog up cells and lead to cell death. Two companies are injecting alpha-synuclein antibodies. One company is injecting fake alpha-synuclein to make the body produce natural antibodies (how the flu vaccine works). 5

6 COULD WE PREVENT THE TOXIC CLUMPS? Another company is testing a drug that stabilizes alpha-synuclein before it can form toxic clumps (called Lewy bodies). This trial is testing in people without Parkinson s, the first step to evaluate safety. If the drug is safe, they ll move to testing in people with PD. 6

7 WHAT DO WE SEE THAT MAY HELP SLOW PARKINSON S? Observations from other studies give us ideas for new therapies. Hypertension Drug (Isradipine): People taking this calcium channel blocker were at lower risk of Parkinson s. Lab tests show isradipine could be neuroprotective. Phase III trial (STEADY-PD) testing same drug for slowing PD Antioxidant Urate Precursor (Inosine): High levels of urate were associated with lower risk of PD or slower progression. Lab tests show inosine could be neuroprotective. Phase III trial (SURE-PD) testing compound the body turns into urate Phase III is the last step before applying for FDA approval. 7

8 WHAT DO WE SEE THAT MAY HELP SLOW PARKINSON S? Observations from other studies give us ideas for new therapies. Nicotine: PD is less prevalent among smokers compared to non-smokers. NIC-PD is testing the impact of nicotine patches on progression. We don t recommend taking up smoking to treat Parkinson s disease. 8

9 WHAT HAPPENED TO NEUROTROPHIC FACTORS? Neurotrophic factors are a family of proteins responsible for the growth of developing neurons and the maintenance of mature neurons. 9

10 WHAT HAPPENED TO NEUROTROPHIC FACTORS? Neurotrophic factors can t be given in a pill, requires brain surgery Only want to grow the neurons killed by disease Scientists are improving delivery methods to: Use enough of the therapy In the right place in the brain For the right amount of time Two studies are ongoing to test these improved methods Also, exercise can help boost neurotrophic factors 10

11 WHY IS IT SO DIFFICULT TO TEST DISEASE-MODIFYING THERAPIES? 1. Clinical trials need hundreds of volunteers. 2. We need a definitive way to choose the right people for studies and to measure the impact of new therapies on disease. Biomarkers: biological characteristics that alert us to risk, onset or progression of disease Scientists are working toward imaging Parkinson s in the brain, measuring proteins in fluid and tissue, and improving clinical measures. 11

12 QUESTIONS & ANSWERS Type your questions in the Q&A box in the middle of your screen. We ll get to as many as we can in this hour.

13 PARTICIPATE IN CLINICAL RESEARCH: foxtrialfinder.org WATCH PREVIOUS WEBINARS: michaeljfox.org/webinars This activity is supported by educational grants from Acadia, Teva and Medtronic. While our generous sponsors make Third Thursdays webinars possible, their support does not influence MJFF s content, perspective or panelist selection.

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