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September 23, 2009 "Michael J. Fox Foundation Commits Up to $5 Million to Help Advance Neurotrophic Factors 'From Promise to Reality' as Parkinson's Treatment 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). full article at: http://www.michaeljfox.org/newsEvents_mjffInTheNews_events_article.cfm?ID=356 Excerpt From the RFA: BACKGROUND In the last several years both preclinical studies and several early Phase clinical trials using neurotrophic factors have showed promise. However, neurotrophic factor therapies have not yet been demonstrated to be safe and efficacious in Phase II clinical trials. A double-blind, placebo-controlled trial of glial cell linederived neurotrophic factor (GDNF) protein infusion sponsored by Amgen failed to meet predetermined clinical endpoints and raised potential safety concerns that resulted in the end of further clinical development despite reports of clinical improvements in some patients. Neurturin (NTN), a GDNF - related factor, has been tested in Phase I and Phase II trials using a viral vector delivery system. While no safety concerns arose in these trials, the Phase II trial failed to meet its primary endpoint at 12 months. However, additional data at later time points show statistically significant, though modest improvements relative to sham controls in patients who continue to be followed. While both the GDNF and NTN trials yielded inconclusive results, many unanswered questions remain about the potential of neurotrophic factors as therapies for PD and vigorous interest remains in further developing neurotrophic factor therapies. MJFF continues to regard neurotrophic factor therapy as a promising approach that deserves additional rigorous investigation in the hopes of advancing the field from promise to reality or at least in definitively determining the viability of the approach PURPOSE The Michael J Fox Foundation for Parkinson’s Research (MJFF) is issuing this Request for Applications (RFA) for investigator-initiated studies to develop neurotrophic therapies for PD. The development of neurotrophic factors as therapies for PD continues to be a priority for MJFF especially since the gene therapy trials done to date demonstrated safety and several Phase I neurotrophic trials have shown signs of efficacy. Through this program, MJFF aims to accelerate the development of neurotrophic factor therapies for PD. The program will fund multi-year (up to three years), potentially multi-million dollar grant awards for milestone driven efforts describing a comprehensive therapeutic development plan. Studies appropriate for this RFA include: ♦ Ambitious projects with a clear plan for accelerating neurotrophic factor therapy through the stages of preclinical and clinical development up to and including Phase II clinical studies. ♦ Therapeutic approaches that include evidence that the neurotrophic factor has the potential for neuroregeneration not merely neuroprotection. Therapeutic development plans should explicitly address how the proposed therapeutic approach overcomes the scientific limitations of previous neurotrophic factor clinical trials (such as inadequate distribution of vector/protein, inappropriate or insufficient target sites in the brain, limited gene expression, inappropriate clinical endpoints, etc.) as well as practical obstacles (such as intellectual property, manufacturing, etc) for advancing therapies through Phase III clinical trials and into widespread clinical use. http://www.michaeljfox.org/funding_programs/Trophic%20Factor%20RFA-Final.pdf ---------------------------------------------------------------------- To sign-off Parkinsn send a message to: mailto:[log in to unmask] In the body of the message put: signoff parkinsn