SECTION 1 : PROGRESS
THE MICHAEL J . FOX FOUNDATION 2022 IN REVIEW
Solving the Parkinson ’ s Puzzle with Disease Markers
Across the entire spectrum of the Parkinson ’ s journey — from prediagnosis through advancing disease — objective , precise measures of Parkinson ’ s disease are needed to enable clear clinical trial outcomes and to improve patient care . Like the legend to a map , such tools would help researchers and clinicians more efficiently navigate every aspect of the therapeutic development ecosystem to accelerate practical outcomes patients and families can feel .
Reading Subtle Changes in Early PD Over the past decade , research has advanced a promising array of new treatments aimed at slowing PD progression . Over that same period , the field has learned that these interventions are best tested in early stages of disease . But changes at this stage can be subtle . To determine whether trials are achieving their intended objectives ( or “ endpoints ”), new and more sensitive measurements must be devised and validated — no small challenge in itself . With the goal of streamlining pathways toward approval for these critical programs , the Foundation is establishing a multidisciplinary advisory committee of experts , including academic and industry researchers , regulatory officials , clinicians and people with Parkinson ’ s , to lead the complex process of defining new and more sensitive endpoints for early PD research within the next three to five years .
A Tool to Streamline Every Alpha-synuclein Trial The importance of alpha-synuclein as a drug target in Parkinson ’ s means that dozens of programs are investigating this critical molecule , with more entering the field almost every month . In the past two years , MJFF funded nine projects to develop a so-called “ alpha-synuclein seeding assay ,” a test that detects and measures the activity of alpha-synuclein protein in biosamples ( e . g ., spinal fluid , skin , saliva ). The test already can diagnose Parkinson ’ s with high accuracy . Additional studies now are under way to optimize it for use in trials and to identify the best kind of sample to use , creating a standardized and transformative tool that can be adopted by research teams for their respective trials — saving research groups years of time and countless dollars of duplicated investment . In addition to developing future iterations of the assay to broaden and expand its impact , MJFF continues to advance its influence through grant funding and distribution of biological samples to qualifying research groups .
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