NEW YORK, NY — The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will provide $1.9 million to support Ceregene Inc.’s Phase II study of CERE-120. This novel gene therapy product, which uses a vector to deliver neurturin, a potent nervous system trophic factor, has shown potential in preclinical studies to slow or stop Parkinson’s disease progression. The funding follows on the announcement of promising early results from Ceregene’s open-label Phase I trial of CERE-120, which also received Foundation support.
“Our partnership with Ceregene bears out two of our top priorities: advancing trophic factor research and moving quickly on clinical research with potential to significantly impact patients’ lives,” said Deborah W. Brooks, president and CEO of The Michael J. Fox Foundation. “We’re enthusiastic about the preliminary safety results from the Phase I CERE-120 trial, with no significant adverse effects seen.”
Foundation funding will be used to provide general support for the Phase II trial and will enable Ceregene’s program, which is headed by Ceregene’s chief operating officer and head of preclinical and clinical development Raymond T. Bartus, PhD, to gather more long-term efficacy and safety data than would otherwise be possible, making the results of the trial more robust. Assuming positive safety and efficacy results, this additional data may also help identify trends that could aid in the design of future trials to test CERE-120’s potential neuroprotective effect in patients.
The MJFF funding complements Ceregene’s own multi-million dollar investment in the study, which will enroll patients at the University of California, San Francisco Medical Center; Rush University Medical Center in Chicago; the University of Pennsylvania; Oregon Health & Science University; Mount Sinai Hospital in New York City; Duke University; the University of Alabama at Birmingham; and Baylor College of Medicine in Houston.
“We are heartened by the Phase I results and pleased that The Michael J. Fox Foundation has continued its support of CERE-120’s clinical testing,” said Jeffrey M. Ostrove, PhD, president and CEO of Ceregene. “We look forward to working with the Foundation’s research staff and scientific advisors, who share our goal of establishing as efficiently as possible whether this therapy will fulfill its promise for Parkinson’s patients.”
This award brings current MJFF investments in neurotrophic factors to $12 million. Neurotrophic factors, also known as trophic or growth factors, have long been considered one of the most promising avenues for Parkinson’s therapies because they promote survival and improve function of neurons. Neurturin is a member of the same protein family as GDNF (glial-derived neurotrophic factor), which has previously been tested in people with Parkinson’s.
The Foundation’s support for Ceregene’s Phase I trial of neurturin was made possible in its entirety by leadership support from The Pioneer Fund, a private family foundation that supports endeavors including medical research.