The Michael J. Fox Foundation Launches New Arm Of Parkinson's Progression Markers Initiative Studying At-Risk Populations In Parkinson's Disease —Study completes enrollment of original 600-member cohort; infrastructure and open-source model are transforming biomarker research—
NEW YORK, April 23, 2013 /PRNewswire-USNewswire/ -- The Parkinson's Progression Markers Initiative (PPMI), a landmark biomarker clinical study, has completed enrollment of its initial 600-member cohort of Parkinson's patients and controls, and will launch additional study cohorts to leverage the existing PPMI infrastructure and evaluate multiple potential biomarkers for Parkinson's disease (PD). The first of these new cohorts launches today and will investigate risk factors for PD that may enable diagnosis before the onset of motor symptoms.
The pre-motor arm of PPMI will enroll participants who do not have Parkinson's disease but do have one of three potential risk factors for PD: a reduced sense of smell (hyposmia); rapid eye movement sleep behavior disorder (RBD); or a mutation in the LRRK2 gene (the single greatest genetic contributor to PD known to date). Research to date indicates that each of these factors can be linked to an increased risk of developing Parkinson's disease, though many people with these conditions do not go on to develop PD. Validating these risk factors and better characterizing their connection to Parkinson's could enable detection of the disease prior to the onset of motor symptoms and open new avenues toward identifying biomarkers — critical tools in the quest for therapies that can slow or stop disease progression.
"If scientists can learn more about the biological processes taking place in people with any of these three risk factors, we may be able to define biomarkers even before typical symptoms begin," said Ken Marek, MD, principal investigator of PPMI and president and senior scientist at the Institute for Neurodegenerative Disorders in New Haven, Connecticut. "Finding a biomarker for PD could mean earlier diagnosis of the disease and lead to new drugs that may delay or even prevent the onset of motor symptoms."
PPMI seeks 10,000 individuals to complete a brief online survey to determine eligibility for the loss-of-smell cohort. Participants in the other groups will largely be enrolled via research centers. This new arm will take place at 23 sites across the world where participants will undergo the same clinical assessments, imaging and collection of biospecimens as the original study.
PPMI's open-source design and infrastructure has opened the door to evaluating multiple potential biomarkers under one umbrella, building on a precedent created by the Alzheimer's Disease Neuroimaging Initiative (ADNI). All PPMI clinical data and characterized biosamples are available in real time, providing researchers around the world with an unprecedented resource to help speed and unify disparate biomarker validation studies. To date, 460 scientists from academia and industry have downloaded PPMI data more than 50,000 times in over 30 countries worldwide, and 21 applications have been made for use of PPMI biospecimens in biomarker research. Initial baseline data from PPMI's original newly diagnosed and control cohorts will be presented this June at the Movement Disorders Society and is expected to be published later this year.
"Lessons learned from Alzheimer's have taught us the importance of pursuing biomarker research concurrent with therapeutic development," said Todd Sherer, Ph.D., CEO of The Michael J. Fox Foundation for Parkinson's Research. "In the third year of PPMI, it is evident that a large-scale biomarker study is not only possible in Parkinson's disease, but is already yielding scientific insights that could help transform the field's pursuit of a cure."
About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest private funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $325 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.
About the Parkinson's Progression Markers Initiative
The Parkinson's Progression Markers Initiative (PPMI) is a $55-million international clinical study sponsored by The Michael J. Fox Foundation for Parkinson's Research (MJFF) and funded by a consortium of 13 industry partners in conjunction with MJFF. Launched in 2010, PPMI 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 in both Parkinson's disease patients and populations at-risk to developing Parkinson's. In April 2013, PPMI completed the recruitment of 400 newly diagnosed PD patients and 200 control subjects and announced the addition of a new arm to investigate potential risk factors of the disease. Using the same infrastructure and protocols, the pre-motor arm of PPMI is evaluating three at-risk cohorts: individuals with decreased sense of smell (hyposmia), people diagnosed with REM sleep behavior disorder (RBD), and those with a LRRK2 genetic mutation (the single greatest genetic contributor known to date).
Researchers interested in applying for access to PPMI data and biospecimens should visit www.ppmi-info.org.
SOURCE The Michael J. Fox Foundation