Gene silencing techniques and induced pluripotent stem cell technology are among the cutting-edge approaches to Parkinson's drug development funded through The Michael J. Fox Foundation's Rapid Response Innovation Awards 2008. As part of its mission to drive transformative treatments and a cure for PD, the Foundation has awarded $1.1 million for 15 high-risk, high-reward Parkinson's disease research projects under the initiative in the first half of the year. Applications are still being accepted, and a total of $2 million in Rapid Response awards is expected by year's end.
Rapid Response helps keep new and novel approaches to Parkinson's disease flowing into the drug development pipeline by allowing researchers to pursue their most exciting ideas in real time. The Foundation accepts Rapid Response proposals on a rolling basis with no deadline, and funding decisions are made within six weeks of application. Awards of up to $75,000 are available for one-year basic, preclinical or clinical research projects in any Parkinson's-relevant arena. The program has met with an enthusiastic response from the research community, both within Parkinson's disease and beyond, since it was first launched in January 2007.
"Rapid Response infuses capital quickly into exciting new ideas that could open up important new avenues of inquiry for Parkinson's disease," said Katie Hood, CEO of The Michael J. Fox Foundation. "Our goal is to provide the funding needed to further 'build the case' for these new concepts, developing the data required before other traditional funding sources can step in."
The program's application process and funding criteria emphasize speed and novelty. Funded projects typically are strong ideas being tested for the first time. Unlike other Foundation initiatives, Rapid Response allows for the submission of applications at any time of year. There is no pre-proposal triage stage, and the standard MJFF application has been shortened to three pages. Additionally, postdoctoral researchers are permitted to apply as principal investigators provided the head of their lab serves as administrative PI.
Among the potentially high-impact Rapid Response projects funded so far this year:
- Asa Abeliovich, MD, PhD, of Columbia University is working to determine whether a gene silencing technique using microRNAs short, noncoding molecules of RNA can be effective in reducing alpha-synuclein, a protein whose aggregation, or clumping, in the brain is a hallmark of Parkinson's pathology.
- Jian Feng, PhD, of SUNY-Buffalo, and Patrick Alfryn Lewis, PhD, of the Institute of Neurology (London, UK) and John A. Hardy, PhD, University College London (London, UK) are conducting two separate investigations using newly discovered induced pluripotent stem cell (iPS) technology to shed greater light on the Parkinson's-implicated genes parkin and LRRK2. Using iPS, the teams are engineering stem cells from skin cells, then using these engineered stem cells to generate human dopamine neurons with or without mutations in the respective genes. Both projects seek to characterize disease mechanisms set off by genetic mutations and to create new models for testing therapeutic approaches that could prevent these events from occurring.
- Rahul Srinivasan, MBBS, PhD, and Henry A. Lester, PhD, of the California Institute of Technology are working to better understand epidemiological findings that have consistently shown smoking may protect against PD. The researchers hope to elucidate the mechanisms by which nicotine may protect dopamine neurons through development and validation of a screening test for small molecules that could increase nicotine receptor expression in the brain.
- Marcus Unger, MD, and Wolfgang Oertel, MD, of Phillips University (Marburg, Germany) want to find better treatments for the digestive problems that dramatically affect Parkinson's patients' quality of life, as well as test the Braak hypothesis, which posits that Parkinson's disease progresses through the body and to the brain in a series of stages starting in the gastrointestinal system. They are examining a possible link between constipation and Lewy body pathology in the gastric lining of people with PD.
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