Charting a New Course to a Biomarker of Parkinson's Disease
Bottom line takeaway: Finding a biomarker is critical to developing treatments that can go beyond symptomatic relief to slow or stop the progression of Parkinson's disease (PD). The Michael J. Fox Foundation (MJFF) is addressing the search head-on and charting an entirely new course in biomarker discovery -- stepping in to take the lead where no one else would.
The potential for patients: Researchers are working hard to find a biomarker for Parkinson's disease, a critical and missing tool in the development of disease-modifying treatments. Reliable and consistent biomarkers for PD would allow scientists to predict, objectively diagnose, and monitor the disease, and to determine which medications work and which do not.
The challenge for researchers: PD is a complex neurodegenerative disorder with many underlying causes that may differ among individuals. For this reason, identifying a biomarker - or set of biomarkers - for PD requires sophisticated methods of measurement targeted at various aspects of the disease. It will take a large scale effort to bring scientists together toward reaching this critical goal.
The MJFF approach: In 2010, MJFF addressed this challenge head-on, launching PPMI, a landmark five-year clinical study to establish a comprehensive set of clinical, imaging and biological data that would define biomarkers of PD progression. To make PPMI a reality, in addition to committing up to $45 million, the Foundation brought together a who's-who of Parkinson's leadership from both academia and industry to conceive, spearhead and fund the study. As the sponsor of PPMI, the Foundation also holds ultimate responsibility for raising awareness of the study and ensuring that its ambitious enrollment goals are met. It is an unprecedented role for a private research funder.
Reasons for optimism: In just two years, PPMI has recruited more than half of the 600 participants required, and the study will expand to 24 sites around the globe in 2012. The study's open-source research model means that well-characterized biosamples and robust clinical data are available to the research community in real time - to speed biomarker validation studies and bring therapeutic breakthroughs closer. Nearly 10,000 data downloads have been made and shared by scientists across the world, all of whom now have access to the largest repository of PD data and biological samples collected to date. Bringing together the research community at large with a groundbreaking data-driven scientific resource, PPMI is serving to drive progress toward biomarker discovery, with the end goal of finding better treatments and a cure for PD.
MJFF research partners: Partners include a consortium of industry players, non-profit organizations and private individuals.
MJFF investment to date: $45 million
Notable quotes: "The role of The Michael J. Fox Foundation in conceiving PPMI and shaping its course has been extremely innovative," says Tom Comery, PhD, Pfizer Neuroscience. "It's truly impressive how the Foundation has brought the community together on this study and Pfizer is proud to be a part of it."