Michael J. Fox Foundation Launches Parkinson's Progression Markers Initiative (PPMI)

- Landmark five-year $40-million global research study aims to identify biomarkers of Parkinson's disease progression, critical tools for drug development -

NEW YORK, Sept. 28 /PRNewswire-USNewswire/ -- 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."

About PPMI

The Parkinson's Progression Markers Initiative (PPMI) is a landmark, five-year international clinical study that aims to find reliable and consistent biomarkers of Parkinson's disease (PD) progression. The study is testing today's most promising biomarker candidates through neuroimaging, the collection of blood, urine, and spinal fluid, and clinical and behavioral tests. Valid measures could allow scientists to predict, objectively diagnose and monitor diseases as well as definitively determine which medications work and which will not. PPMI is sponsored by The Michael J. Fox Foundation for Parkinson's Research and funded by a consortium of industry partners and individual donors. To learn more or volunteer to participate in the study, visit www.michaeljfox.org/PPMI or call (877) 525-PPMI.

About The Michael J. Fox Foundation

The Michael J. Fox Foundation http://www.michaeljfox.org is dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson's today. MJFF has funded almost $200 million in research to date.

Fact Sheet: Parkinson's Progression Markers Initiative (PPMI)

September 28, 2010

What is PPMI?

• The Parkinson's Progression Markers Initiative is a landmark, five-year, international clinical study to identify biomarkers for the progression of Parkinson's disease.
• The study will follow 400 newly diagnosed Parkinson's patients who have not yet started taking medication and 200 individuals who do not have PD.
• Researchers will study images of participants' brains, biological samples (blood, urine and spinal fluid) and clinical/behavioral tests.
• The Michael J. Fox Foundation for Parkinson's Research is sponsoring PPMI, which is expected to cost $40 million. It will be funded by the Foundation and a consortium of industry partners and generous donors, including Pfizer Inc., GE Healthcare, and Mrs. Lily Safra, a member of the Foundation's Board of Directors.

What is a biomarker, and why do we need to find one for PD?

• A biomarker is a substance, process or characteristic in the body that is associated with the risk, presence or progression of disease. There is currently no known biomarker for PD.
• It is hoped that a biomarker for PD will provide scientists with a critical tool to predict, objectively diagnose and monitor the disease as well as definitely determine which medications work and which do not.
• A biomarker will aid academic and industry researchers in the quest for disease modifying therapies that can slow or stop the progression of PD. No such drug has yet been found.

What are the implications for industry?

• Drug companies would like to develop disease-modifying treatments, but attempts to do so have been both costly and difficult.
• To bring a new central nervous therapy to market requires an investment of over $1 billion (Adams & Brantner, Health Affairs 25 no. 2 (2006) 420-428, Exhibit 5: Probability of Market Entry, Durations, and Costs for New Drugs, by Disorder and Primary Indication) and takes over nine years. A biomarker could dramatically reduce both the cost and time of development.
• Approximately one million Americans currently have PD.  An estimated five million people around the world are living with PD. Current annual U.S. sales of PD therapies are estimated at $800 million (Health Advances, 2006); that could increase to $2-3 billion (Health Advances, 2010) with the advent of a disease-modifying therapy.

What is the significance of PPMI for research?

• PPMI will establish a scientific model for testing potential biomarkers for Parkinson's disease. This is the first time that samples and data have been collected in a population of sufficient size over enough time to draw meaningful conclusions about the onset and progression of PD.
• Through PPMI's "open source" research model, the data and samples will be made available to qualified researchers outside the study to help spark further innovation and collaboration, in order to develop new, more effective treatments more quickly.
• The Foundation is sponsoring PPMI now, because for the first time, the Parkinson's field has amassed enough biomarker leads and initial data to justify the development of a comprehensive biomarker validation infrastructure.

Where is the research taking place?

• Scientists at 18 locations throughout the United States and Europe will examine patients and collect data; the data will be stored at seven Cores throughout the country.

Sites Currently Accepting Volunteers:

The University of Alabama at Birmingham, Birmingham, Alabama

Arizona Parkinson's Disease Consortium, Sun City, Arizona

Baylor College of Medicine, Houston, Texas

Institute for Neurodegenerative Disorders, New Haven, Connecticut

Northwestern University, Chicago, Illinois

The Parkinson's Institute and Clinical Center, Sunnyvale, California

Boston University, Boston, Massachusetts

Oregon Health & Science University, Portland, Oregon

University of Pennsylvania, Philadelphia, Pennsylvania

Additional Sites in the Following Cities Will Join the Study:

U.S. Cities

Atlanta, Georgia

Tampa, Florida

Baltimore, Maryland

Rochester, New York

Seattle, Washington

European Cities

Innsbruck, Austria
Kassel/Marburg, Germany
Tuebingen, Germany

Naples, Italy

PPMI Study Cores:

Bioanalytics Core: University of Pennsylvania School of Medicine

The Bioanalytics Core will perform some of the biomarker verification studies identified by the Steering Committee and will be working closely with the PPMI Biorepository in monitoring and performing quality control on collected samples.

Bioinformatics Core: Laboratory of Neuro Imaging (LONI), Department of Neurology, UCLA School of Medicine

The Bioinformatics Core has designed and will maintain the PPMI study database. This Core is also responsible for the programming of the study Web site.

Biorepository Core: Coriell Institute for Medical Research

The PPMI Biorepository will receive, store and distribute biospecimens collected throughout the course of the study.

Clinical Core: Clinical Trials and Coordination Center (CTCC), University of Rochester

The Clinical Core is responsible for site management, as well as the collection, tracking and performance of quality control on clinical data.

Genetics Core: Laboratory of Neurogenetics, National Institute on Aging/NIH

The Genetics Core will analyze DNA collected from study subjects.

Imaging Core: Institute of Neurodegenerative Disorders

The Imaging Core will receive, aggregate and perform quality control on all imaging data in the study. This Core will also perform preliminary analyses of imaging data, as directed by the Steering Committee.

Statistics Core: Clinical Trials Statistical and Data Management Center, University of Iowa

The Statistics Core will lead the statistical analyses that will be performed on the PPMI data and samples.

DTI Analysis Core: Northern California Institute for Research & Education, University of California San Francisco, Department of Radiology & Biomedical Imaging

The DTI Analysis Core will lead the analyses that will be performed on the MRI/DTI data.

For More Information about PPMI, Visit www.michaeljfox.org/PPMI or call (877) 525-PPMI.

ABOUT THE MICHAEL J FOX FOUNDATION (MJFF) The Michael J. Fox Foundation is dedicated to finding a cure for Parkinson's disease through an aggressively funded research agenda and to ensuring the development of improved therapies for those living with Parkinson's today.  The Foundation has funded nearly $200 million in research to date.

ABOUT PPMI The Parkinson's Progression Markers Initiative (PPMI) is a landmark, five-year international clinical study that aims to find reliable and consistent biomarkers of Parkinson's disease (PD) progression. The study is testing today's most promising biomarker candidates through neuroimaging, the collection of blood, urine, and spinal fluid, and clinical and behavioral tests. Valid measures could allow scientists to predict, objectively diagnose and monitor diseases as well as definitively determine which medications work and which will not. PPMI is sponsored by The Michael J. Fox Foundation for Parkinson's Research and funded by a consortium of industry partners and individual donors. To learn more or volunteer to participate in the study, visit www.michaeljfox.org/PPMI or call (877) 525-PPMI.

SOURCE Michael J. Fox Foundation