NEWYORK, NY — The Michael J. Fox Foundation has committed up to $5 million for research to successfully translate neurotrophic factor advances from pre-clinical and early-phase clinical work into a practical, patient-relevant treatment for Parkinson’s disease. The funding will be awarded under a new Directed LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions) initiative.
The Foundation’s intention is to identify and drive ambitious projects with a clear plan for accelerating trophic factor therapy through the stages of preclinical and clinical development up to and including Phase 2 clinical studies. Also of interest are therapeutic approaches that include evidence that the trophic factor being studied holds potential for neuroregeneration (helping the brain rejuvenate sick cells) and not merely neuroprotection (protecting existing neurons from death).
Neurotrophic factors (also known as trophic factors or growth factors) are specialized proteins that protect and nourish neurons in the brain, including the dopamine neurons that die in Parkinson’s disease. They are a high-priority therapeutic target for MJFF, which has committed approximately $20 million to their development to date, because they have shown potential to slow or stop the progression of Parkinson’s in the brain — something no currently available treatment has been proven to do.
In the last several years, preclinical studies and several early-phase clinical trials in neurotrophic factors have shown promise to revolutionize the treatment of PD. Research increasingly indicates that trophics are safe for use in people, and several Phase 1 neurotrophic clinical trials have shown signs of efficacy. But results from all previous trials have been ultimately inconclusive, and no trophic-based approach has yet been demonstrated to be both safe and efficacious in a Phase 2 clinical trial.
“While many unanswered questions remain about the potential of neurotrophic factors as therapies for PD, MJFF continues to regard neurotrophic factor therapy as a promising avenue,” said Todd Sherer, PhD, vice president of research programs at MJFF. “We believe the approach deserves additional rigorous investigation in hope of advancing the field from promise to reality — or at least definitively determining its viability.”
This Directed LEAPS program is open to academic and industry researchers. Pre-proposals are due November 18, 2009. Funding is anticipated in June 2010. The full RFA and detailed information on submitting an application are available at www.michaeljfox.org/research.