The Michael J. Fox Foundation Annual Report 2017 – Roadmaps for Progress - Page 6

The Michael J. Fox Foundation 2017 Annual Report Annual Letter from the CEO and the Co-Founder Todd Sherer, PhD, (right) with David Standaert, MD, PhD, at the annual PD Therapeutics Conference Debi Brooks with Michael J. Fox at an MJFF salon sharing research progress Dear Friend, Understanding Parkinson’s disease is the starting point of every effort to accelerate the cure. Years of work spent uncovering Parkinson’s secrets — defining the highly variable patient experience, shedding light on genetic origins of disease, mapping molecular pathways — are now paying off in a tangibly quickening tempo of progress. Researchers are increasingly linking pathology to clinical symptoms (and vice versa) to inform therapeutic target and biomarker identification. 4 This has positioned drug makers to make rapid inroads toward treatments with potential to slow, stop and perhaps even prevent Parkinson’s disease (PD) symptoms and cell loss. 2017 saw the launch of the first precision medicine clinical trials targeting genetically defined forms of Parkinson’s disease (LRRK2 and GBA). Numerous therapies acting on other pathologies, or auguring improved management of disabling symptoms, continued through clinical trials, pushing ever closer to market. Multiple consortia and countless investigators worked in tandem to fill in ever-larger sections of the Parkinson’s puzzle, characterizing cellular pathways and linking outward dysfunction to underlying disease progression. A cornerstone of these efforts, our Parkinson’s Progression Markers Initiative (PPMI), is influencing clinical trial design and vastly improving understanding of the natural history of disease. Today, researchers are maximizing the value of precious PPMI samples through cutting-edge “omics” analyses, techniques that are speeding 2017 Progress in Drug Development the identification of novel biomarker candidates and the nomination of improved therapeutic targets. And, as demonstrated in part by the willingness of patients and families to participate in PPMI, The Michael J. Fox Foundation (MJFF) also has invested in building more onramps for engaging the PD community. In October 2017, we launched Fox Insight (foxinsight.org) — our online clinical study that is gathering patient-reported data on experience of PD from, at time of printing, nearly 20,000 people with PD and their loved ones. The study aims to enroll hundreds of thousands of people amplifying the patient voice in Parkinson's research. In collaboration with 23andMe, Fox Insight is also capturing genetic data for continued biological discovery, matching genotype to phenotype. While 2017 was a year full of progress and promise in Parkinson's research, much work remains to reveal the molecular fingerprint of the disease, draw lines between pathological bad actors and physical manifestation, and The Parkinson’s drug development pipeline is abuzz with activity, and new therapies to treat motor and non-motor symptoms are market-bound. Two new Parkinson’s drugs hit pharmacy shelves in 2017. Newron’s Safinamide (Xadago), an add-on therapy for those dealing with persistent symptoms despite levodopa, received U.S. Food and Drug Administration (FDA) approval in March. And the first drug specifically indicated for levodopa-induced dyskinesia — Adamas’ Gocovri, an extended-release formulation of amantadine — arrived in August. MJFF enabled the drug on its path by providing strategic leadership and $1 million in funding for the development of a rating scale instrumental to testing dyskinesia drugs in transform rapidly accumulating insights into reliable measures and curative therapies. This information is critical to speed development of therapeutics already under way as well as those still to be discovered. On the following pages, we share the Foundation’s roadmap approach, designed to enable and structure this work in order to accelerate knowledge turns.