Last night, Michael J. Fox returned to television as the star of his own sitcom after more than two decades living with Parkinson’s disease.

Fox’s decision to return to primetime has injected Parkinson’s into the national conversation — a conversation already transformed by The Michael J. Fox Foundation for Parkinson’s Research (MJFF), which the actor launched in 2000 with the exclusive goal of funding research to speed a cure for the disease.

“What I had in mind was an organization built for speed, eschewing bureaucracy and taking an entrepreneurial approach toward helping researchers find a cure for Parkinson’s,” says Michael J. Fox. “There was no existing blueprint for accomplishing what we wanted to do. We were faced with the challenge of inventing a new system.”

Parkinson’s disease is estimated to affect one in 100 people over age 60. It is the second-most common brain disorder after Alzheimer’s and the 14th leading cause of death in the United States. The average age of onset is 60, though some people are diagnosed at age 40 or younger. There is no cure, and available treatments are inadequate to patients’ medical needs: They address only some of the symptoms and bring disabling side effects of their own.

“Michael J. Fox’s return to television has created an opportune moment to consider how much work remains to be done in the realm of neurodegeneration research,” wrote MJFF CEO Todd Sherer, Ph.D., in an op-ed published by Scientific American on September 25. “For every patient, a community is affected, as the impact of PD ripples to loved ones and caregivers. This is a global problem, but one that we can and must solve.”

Largest Nonprofit Funder of Parkinson’s Disease Research

The Michael J. Fox Foundation grew quickly from startup to the world’s largest nonprofit funder of Parkinson’s research, and has made grants totaling more than $350 million to research teams all over the world (about 30 percent of funds go to international teams). In November 2008 The New York Times called MJFF “the most credible voice on Parkinson’s research in the world.”

The Foundation typically allocates donor capital within a few months of receiving donations, eschewing an endowment or large investment reserve to quickly push research to the next level. Since inception, 89 cents of every dollar spent by MJFF has gone directly to high-impact research programs. Genetic Engineering & Biotechnology News called MJFF a “beacon of efficiency” after the Foundation placed first on their list of 20 grant-giving disease foundations in May 2013.

Due in no small part to the Foundation’s efforts, more new Parkinson’s drugs are in development today than ever before. A high-risk, high-reward model drives investment in early-stage research, getting innovative new projects off the ground to make them attractive to larger-scale partners. Earlier this month, biotechnology companies Amicus Therapeutics and Civitas Therapeutics, Inc., announced follow-on investments to projects kick-started with MJFF funding. Additional support for these projects — around a disease-modifying drug target and improved delivery of symptomatic treatment — will mean faster development and delivery of therapies that will improve the lives of Parkinson’s disease patients.

Patient Engagement to Accelerate Discovery and Development

In addition to funding promising studies, MJFF problem-solves to address operational challenges in the landscape of biomedical research. One roadblock to drug discovery and development is the lack of reliable and validated Parkinson’s biomarkers. A biomarker — a substance, process or characteristic in the body associated with the risk or presence of disease or that changes over time in a way that can be linked to disease progression — can help diagnose the disease sooner, track its progression and test the effects of new drugs.

The MJFF-sponsored landmark Parkinson’s Progression Markers Initiative (PPMI) launched in 2010 with 24 clinical sites around the world capturing and analyzing standardized Parkinson’s disease patient and healthy control data to identify biological markers of the disease. The first significant PPMI results were published last month in JAMA Neurology, where investigators reported differences between Parkinson’s patients and controls in spinal fluid protein levels. Research continues into these and other potential biomarkers, and PPMI has expanded to study individuals with Parkinson’s risk factors to try to characterize the disease before the onset of motor symptoms. The hope is that such understanding will lead to a preventive therapy for Parkinson’s.

To identify biomarkers and test new drugs, research needs volunteers; 85 percent of clinical studies are delayed due to slow volunteer enrollment. MJFF created Fox Trial Finder, an online clinical trial matching tool to connect interested Parkinson’s patients and healthy controls to Parkinson’s disease studies they may be eligible for and interested in. With more than 20,000 registrants and 300 active trials in the United States, Canada, United Kingdom, Ireland and Australia, Fox Trial Finder is launching this fall in five additional European countries: Germany, France, Spain, Italy and Austria.

think/able Campaign Collects Messages for Michael

To mark Michael J. Fox’s return to television, the Foundation has launched the think/able campaign, which celebrates the power of optimism and proactivity to overcome challenges and meet our goals. Now through the end of October, the project is collecting messages to Michael from the community. All are welcome to submit a message using the form available on the Foundation’s Web site at www.michaeljfox.org/thinkable.

Source: PR Newswire

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