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Repositioning RFA, A Potential PD Vaccine and PPMI Update

In the fall 2011 issue of our newsletter, Accelerating the Cure, Nate Herpich, MJFF's associate director of research communications, provided updates on some of our funding, including: drug repositioning, a potential PD vaccine and PPMI. Check out the newsbriefs below or click here to read the rest of the fall issue.



Reducing the Drug Development Timeline: Repositioning RFA

Over the past year, The Michael J. Fox Foundation has ramped up its interest in repositioning. This is the process of testing a drug that is already FDA-approved or that has been proven safe in a clinical trial for a certain indication, to determine whether the drug might be efficacious in the treatment of another therapeutic area, like Parkinson’s disease (PD). “Repositioning can drastically reduce the timeline for getting a drug into clinical testing for PD, making an impact on patients’ lives that much sooner,” says Kuldip Dave, PhD, associate director of research programs at MJFF.

While MJFF has supported repositioning projects before, its first-ever repositioning specific Request for Applications (RFA) launched in fall 2010. MJFF announced the results of this RFA in July: six researchers with awards totaling $2.4 million. Three additional awards were granted in August through other programs, bringing the total funding for repositioning to $3.4 million. Several of the research teams are working with drugs that could offer protection to the brain cells that die in Parkinson’s disease, including a tuberculosis vaccine and the antidepressant duloxetine (marketed as Cymbalta). Others are testing symptomatic therapies, including a compound used to treat Attention Deficit Hyperactivity Disorder (ADHD).

A Potential Vaccine to Slow Parkinson’s Disease Progression

This summer, MJFF awarded funding for a first-of-its-kind therapeutic approach to Parkinson’s disease: a vaccine targeting alpha-synuclein, a protein implicated in Parkinson’s disease. The vaccine aims to slow or stop the progression of Parkinson’s, something no current treatment can do.

Vienna, Austria-based company AFFiRiS AG will conduct a clinical study of the safety and efficacy of its vaccine candidate PD01. The hope is that this agent will simulate the production of antibodies that bind to alpha-synuclein, clearing it from the brain and slowing disease progression. Alpha-synuclein is a high-priority target for MJFF, largely because there is compelling evidence that it may play a role in both genetic and idiopathic cases of PD. It is also the major component in the Lewy bodies that are the pathological hallmark of Parkinson’s.

The trial, led by AFFiRiS chief medical officer Achim Schneeberger, MD, will engage 24 subjects with mild Parkinson’s disease over two years at a single clinical site in Austria. “Cautious optimism is called for,” says Jamie Eberling, PhD, MJFF’s associate director of research programs, “but it’s important to bear in mind that PD01 is still in the early stages of clinical testing.”

On the Path to Finding Biomarkers: PPMI Update

Since MJFF launched the Parkinson’s Progression Markers Initiative (PPMI) more than a year ago, the project has grown in scope, and it continues to expand its presence across the United States and Europe. Nine industry funders, including leading pharmaceutical and biotech companies, have partnered with MJFF to support this work, and researchers and clinicians across the world have begun to analyze the data and biological samples that could unlock clues to the onset and progression of Parkinson’s disease. In a move to make PPMI a truly global study, in August MJFF announced a collaboration with the Shake It Up Foundation Australia to bring PPMI down under, increasing our chances to quickly identify universally relevant biomarkers.

Of 21 planned clinical sites in the United States and Europe, 19 are now active, with two more in the EU set to begin enrolling participants soon. 179 individuals are actively enrolled at PPMI sites, including 101 newly diagnosed PD participants, and 78 control participants who do not have PD. Another 45 volunteers are pending enrollment. Samples and data collected in PPMI are available to the scientific community at large in real time for promising biomarker validation studies. Scientists using the initiative’s resources agree to report back to PPMI with findings and data, enabling future researchers to build on their work to help speed promising PD biomarker validation efforts worldwide.

You, too, can help with these efforts. PPMI continues to recruit and must find at least 400 people who are newly diagnosed with PD, and 200 controls. If you or someone you know might want to participate in PPMI, visit to learn more about our study and find the nearest site.


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