Penn Medicine selected as study site for PPMI

University of Pennsylvania School of MedicineSep 30 2010

$40 million, 5-year observational clinical study, sponsored by Michael J. Fox Foundation, seeks biomarkers of Parkinson's disease

The Penn Medicine Parkinson's Disease and Movement Disorders Center announced that it is one of 18 official study sites for the Parkinson's Progression Markers Initiative (PPMI), a landmark observational clinical study sponsored by The Michael J. Fox Foundation, which will use a combination of advanced imaging, biologics sampling and behavioral assessments to identify biomarkers of Parkinson's disease progression. Enrollment of 20 patients and 10 controls at the Penn Parkinson's Disease and Movement Disorders Center is expected to begin in October 2010 and will continue for approximately two years.

"PPMI will provide invaluable data on the early Parkinson's disease process, laying the critical groundwork for breakthrough therapies that are aimed at the underlying mechanisms of disease," said Matthew Stern, MD, professor of Neurology at the University of Pennsylvania School of Medicine and director of the Penn Parkinson's Disease and Movement Disorders Center. "Penn Medicine has been at the forefront of Parkinson's disease research and care for years. It is deeply meaningful to have been selected as a PPMI site and to have our commitment to speeding Parkinson's solutions recognized."

Said Michael J. Fox: "This is an ambitious undertaking, no doubt. But nothing worth having comes easily. Everything we've learned up to now, the partnerships we've worked to forge, the results of research we've funded - it's all put us in position to launch this effort. We're ready to roll up our sleeves and, hopefully, get this done."

"From the patient's perspective, participation in PPMI is quite distinct from a typical clinical trial. Rather than helping to test a single therapy, patients and health controls who participate in PPMI will be building resources to accelerate development of a multitude of treatments for years to come," said Andrew Siderowf, MD, associate professor of Neurology at the University of Pennsylvania School of Medicine, who is a member of the PPMI study steering committee and a site investigator at Penn. "We are certainly asking patients and controls to do a lot in this study, but the product of their efforts will be a cornerstone of the next generation of PD research."

John Q. Trojanowski, MD, PhD, director of the Penn Udall Center for Parkinson's Research and Leslie M. Shaw, PhD, professor of Pathology and Laboratory Medicine at the University of Pennsylvania School of Medicine will lead the PPMI Bioanalytics Core. They will be responsible for analyzing samples collected from all trial participants, using a test they designed, standardized and validated to measure levels of specific biomarkers in spinal fluid. Drs. Trojanowski and Shaw, who also serve as co-leaders of the Biomarker Core for the Alzheimer's Disease Neuroimaging Initiative (ADNI), will provide additional experience to consult with the PPMI Biorepository Core, to ensure the samples are collected and stored in a standardized way. In addition, they will be part of a collaborative group evaluating any novel diagnostic biomarkers that emerge in the course of the trial, to see if they can and should be integrated into the PPMI study.

"This bold cutting edge study addresses an important unmet need for development of better Parkinson's disease diagnostics that will have as profound an impact on the diagnosis and treatment of Parkinson's disease as the ADNI study is having now on Alzheimer's disease," said Dr. Trojanowski and Dr. Shaw. "Now is the time to move full speed ahead with PPMI. We applaud The Michael J. Fox Foundation for its vision and leadership with this project."

A biomarker could be any objectively measurable physical characteristic associated with the presence of disease (diagnostic or risk marker) or any characteristic that changes over time in a way that can be tied to the progression of disease (progression marker).

"There is no doubt that finding a biomarker is critical to the development of next-generation therapies, and that the lack of this tool is among the most critical issues facing the PD research field," said Katie Hood, CEO of The Michael J. Fox Foundation. "MJFF has funded biomarker discovery efforts for years. Now we are poised for a concerted, unified effort that will take these discoveries to the next level."

The lack of a PD biomarker impedes PD diagnosis and treatment, and also critically stalls the development of improved therapies, particularly therapies to slow or stop the progression of PD, something no currently available treatment can do. Clinical trials of new, potentially disease-modifying Parkinson's treatments are at risk of yielding inconclusive results, because there is no way to measure the effects of those treatments objectively.