NEWYORK, NY — As of mid-2009, The Michael J. Fox Foundation for Parkinson’s Research has already funded over $1 million in high-risk, high-reward approaches to Parkinson’s therapeutic development through its groundbreaking Rapid Response Innovation Awards. Projects include investigations of the safety and efficacy of an FDA-approved drug for kidney disease to treat Parkinson’s; characterization of a tenuous link between PD and certain strains of influenza virus; and a proof-of-principle approach to refine deep brain stimulation (DBS) into a potentially more effective symptomatic therapy.
Rapid Response, one of the Foundation’s Edmond J. Safra Core Programs for PD Research, earmarks up to $2 million annually to support research with little to no existing preliminary data, but potential to crack open entirely new ways of understanding or treating Parkinson’s disease. The program has no deadline, instead accepting researchers’ proposals on a rolling basis throughout the year in order to fund truly novel thinking whenever inspiration strikes.
“Rapid Response enables us to assess the best new ideas in real time, quickly vet their potential, and move the most promising ones to the next level fast,” said Katie Hood, CEO of MJFF. “This reflects our commitment to keep fresh ideas flowing into the Parkinson’s therapeutic pipeline, as well as our recognition that when great research ideas have to wait for a program deadline, so do PD patients — a status quo we are determined to improve on.”
Highlights of the 2009 program include:
- Curt R. Freed, MD, of the University of Colorado has discovered that phenylbutyrate, a drug already approved by the U.S. FDA to treat kidney disease, prevents brain deterioration in pre-clinical models of Parkinson’s. The drug may turn on a protective gene called DJ-1 in the brain. Dr. Freed is studying 12 people with recently diagnosed PD to determine if the drug can increase DJ-1 levels and set the stage for determining whether phenylbutyrate may slow disease progression.
- Richard Smeyne, PhD, of Saint Jude Children's Research Hospital will test the hypothesis that the virulent H5N1 (“bird flu”) strain of influenza virus can play a role in inducing parkinsonism. There is significant literature, mostly based on cases from the Spanish Influenza outbreak of 1918, documenting Parkinson’s disease following exposure to flu virus. The researchers hypothesize that flu can induce aspects of PD including cell loss, as well as to show how viruses access the nervous system.
- Wassilios Meissner, MD, PhD, of University Hospital Bordeaux (France) and Peter Tass, MD, PhD, of the University of Cologne (Germany) want to update deep brain stimulation surgery, taking advantage of new discoveries about the mechanisms of PD to expand DBS’s benefit to a wider range of PD patients. With MJFF funding, they are working in a pre-clinical model of PD to assess the practical potential of modifying abnormal synchronization in the brain’s basal ganglia region — an approach that, according to preliminary theoretical studies, could result in functional improvements for PD patients.
The Foundation has funded 20 Rapid Response Innovation Awards in 2009, and a total of $6,081,627 in awards since the program was first launched. Some Rapid Response projects from prior years have gone on to garner major additional funding to support the next stage of research, including clinical trials. These include D. James Surmeier, PhD’s 2007–2008 investigation of approved high-blood pressure drug isradipine as a neuroprotective approach to PD, which is currently being tested with MJFF support in a Phase 2 controlled clinical trial.