Michael J. Fox Foundation Awards $4.6 Million to Help Advance Neurotrophic Factors as Transformative Parkinson's Treatments
NEW YORK, NY — The Michael J. Fox Foundation for Parkinson’s Research today announced $4.6 million in awards to support two promising studies of neurotrophic factor therapies for Parkinson’s disease. The projects, funded under a directed LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions) initiative, bring together leading investigators to build on the knowledge and experience gained from previous trials of neurotrophic factors to help speed this avenue of research toward better treatments for people living with PD.
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. Preclinical studies and several early-phase clinical trials of trophic factors have shown potential to slow or stop the progression of Parkinson’s – a key unmet need for PD patients. But the results from Phase 2 clinical trials have yet to demonstrate the efficacy of neurotrophic factor therapies. A complication in developing trophic-based therapies is the inability of these large proteins to cross directly into the brain, requiring innovative methods for drug delivery.
“MJFF continues to believe in the potential of neurotrophic factors as transformative therapies for people living with Parkinson’s disease,” said Todd Sherer, PhD, vice president of research programs at MJFF. “The Foundation’s new approach to this area of research is to develop better methods to deliver trophic factors to the appropriate brain regions at the appropriate doses. And that’s exactly the focus of the two projects that MJFF is supporting.”
One of the awardees, the biopharmaceutical company Ceregene, Inc. will be conducting a Phase 2 clinical study evaluating CERE-120, a gene therapy product which aims to deliver the neurotrophic factor neurturin to dying dopamine neurons in the brain. Ceregene’s new clinical study follows an earlier Phase 2 trial, also supported by MJFF, which failed to show a benefit of CERE-120 over placebo on the primary endpoint (Unified Parkinson’s Disease Rating Scale) after 12 months. However, continued follow up of blinded data from the trial participants showed a statistically significant treatment effect for CERE-120 at 18 months and, at that time, MJFF partnered with Ceregene to further analyze trial data. Based on the findings that suggested inadequate distribution of CERE-120 to the area of the brain affected by PD, Ceregene has enhanced the method of delivery and dosing regimen of its gene therapy for use in continuing clinical studies.
The second award supports a collaborative effort between Biovail Laboratories International SRL and MedGenesis Therapeutix, Inc. to develop a different neurotrophic factor, GDNF (glial cell line-derived neurotrophic factor), as a potential PD therapeutic. Although earlier clinical studies of GDNF have shown promise, no trial has conclusively demonstrated the safety and efficacy of this trophic factor as a Parkinson’s treatment. Biovail and MedGenesis are working on an effective method of delivering GDNF to targeted areas of the brain, addressing a key limitation of previous studies. MJFF’s support will help advance this work, bringing the potential benefits of GDNF closer to patient relevance.
Neurotrophic factors are a high-priority therapeutic target for MJFF, which has committed approximately $20 million to their development to date. For more information about the projects announced today, including grant abstracts and researcher bios, visit www.michaeljfox.org.