Michael J. Fox Foundation launches PPMI clinical study to identify Parkinson's disease biomarkers

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The Michael J. Fox Foundation has launched the Parkinson's Progression Markers Initiative (PPMI) (www.michaeljfox.org/PPMI), the first-ever large-scale clinical study exclusively focused on identifying and validating Parkinson's disease biomarkers.

A biomarker is required to efficiently test potentially life-transforming new drugs that could slow or stop the progression of Parkinson's disease — something no PD treatment on today's market can do. A biomarker of Parkinson's has not yet been found.

"Currently, Parkinson's patients have access only to treatments that temporarily alleviate symptoms. If a biomarker is found, researchers will have a vitally needed tool in the quest for disease-modifying therapies that can do more than simply mask symptoms of the disease," said Katie Hood, CEO of The Michael J. Fox Foundation. "While there is no guarantee that validated biomarkers will result from the PPMI study, their importance to therapeutic progress requires that we accept this risk."

The five-year study is sponsored by The Michael J Fox Foundation and is expected to cost $40 million over five years. It will be funded by the Foundation with a lead gift from Mrs. Lily Safra, a long time friend, partner, and board member of MJFF, and through the generous support of industry partners including Pfizer and GE Healthcare.

PPMI will be led by Principal Investigator Kenneth L. Marek, MD, President and Senior Scientist, Institute for Neurodegenerative Disorders, New Haven, Connecticut.

"With PD progression biomarkers in hand, it will be possible to establish objective endpoints for clinical trials of disease-modifying Parkinson's treatments," said Dr. Marek. "Endpoints are concrete ways to demonstrate that a candidate therapy is, or is not, slowing disease course in PD patients, as opposed to simply treating disease symptoms. Without a biomarker, this sort of evidence-based decision-making is much more difficult."

The study will be carried out at 18 sites in the United States and Europe (see list in fact sheet below). It will track 400 people newly diagnosed with PD and 200 who do not have the disease. Recruitment of study volunteers is now under way at six sites, with all sites expected to be recruiting by year-end.

An observational study as opposed to an interventional trial, PPMI will not test any experimental drug. Participants will be contributing to a large body of data and biological specimens whose aim is to further biomarker research.

PPMI will make biological samples and rich clinical data from a single, large and well-characterized cohort available to qualified researchers around the world. The goal of the collaboration is to help increase the pace of biomarker validation and clinical testing as well as accelerate the pace of discovery. Researchers interested in following PPMI's progress in real time should visit the study's scientific Web site at www.ppmi-info.org.

"I believe that we are going to find the cure for Parkinson's disease — but we all have to work together to make it happen," said Michael J. Fox. "There's something you can do to help change the lives of millions of people."

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