This week, The Wall Street Journal reported from TEDMED on an issue close to The Michael J. Fox Foundation’s heart: “Bridging the Gap Between Basic Research and Patient Care.”

The Journal highlighted remarks from National Institutes of Health head Francis Collins, who provided colorful illustrations for how to accelerate translational research in drug development, as he called for the need to “build a bridge across this yawning gap” in order to bring novel drugs to the people that need them, faster.

Imagining the gulf between basic science and applications as a body of water, Collins said linking them wouldn’t be like building the Golden Gate Bridge. Continue reading ““Building a Bridge Across a Yawning Gap”” »

This week, several news outlets including The Wall Street Journal highlighted a trend toward repositioning drugs that are already FDA-approved to determine whether they might be efficacious in the treatment of another therapeutic area. One such drug, already available to treat multiple sclerosis, has entered a Phase II trial to determine its ability to also treat Lou Gehrig’s disease, or amyotrophic lateral sclerosis (ALS).

In 2012, MJFF made Repositioning Drugs for PD one of its recurring programs, following a 2011 in which the Foundation funded nine grants for $3.4 million in repositioning drugs that could benefit people with PD.  By identifying therapies that are already clinically available, MJFF hopes to reduce the time and cost it takes to bring PD potential drugs to market. Continue reading “Trend Toward Drug Repurposing Highlighted in The Wall Street Journal” »

This week, the MJFF research staff reflected on some of the projects they worked on in 2011 that they believe could have the greatest impact on the Parkinson’s disease (PD) patient community.  Read on to find out directly from Foundation team members about some of the work being done to speed progress toward a cure.

“I’m enthusiastic about the progress made in 2011 on PPMI.  The study has now enrolled over 280 individuals to participate in the study, and data have been already downloaded more than 4,000 times by scientists in the research community at large to conduct independent studies toward verifying biomarkers for PD.   MJFF also helped make a specific lab test available to researchers that could help to measure the protein alpha-synuclein as a potential biomarker for the disease – a huge step forward.  Thanks to such advancements, I am hopeful that PPMI will in the future have a major impact on drug development for PD, tangibly benefiting those living with the disease.” – Mark Frasier, PhD, director of research programs

Continue reading “MJFF Research Staff Reflects on Some of 2011′s Big Impact Projects” »

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.

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RESEARCH NEWSBRIEFS

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 www.michaeljfox.org/PPMI to learn more about our study and find the nearest site.