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Michael J. Fox Foundation Announces Funding for Ceregene Phase I Gene Therapy Clinical Trial

NEWYORK and SAN DIEGO— The Michael J. Fox Foundation for Parkinson’s Research (MJFF) today announced a three-year grant of approximately $740,000 to Ceregene, Inc., a San Diego-based biotechnology company. The grant will help fund Ceregene’s Phase I clinical study of CERE-120, a novel gene therapy product that has shown potential in pre-clinical testing to slow or stop the progression of Parkinson’s disease by using a vector to deliver neurturin, a potent nervous system growth factor.

While the primary goal of any Phase I clinical study is to demonstrate safety, Ceregene will also measure the efficacy of CERE-120 through brain imaging studies and standardized Parkinson’s tests. MJFF support will significantly enhance the speed and depth of this data collection, allowing for more regular testing of a wider range of neurological functions than would otherwise be possible. Each patient will undergo a PET scan and a full battery of neurological tests every three months (in addition to regular visits to the clinic for more routine procedures such as blood tests). Assuming successful results in the first study, measurements from the Phase I study will lead to more efficient planning of a larger Phase II study that will gather more detailed data on both safety and efficacy.

“The Phase I trial of CERE-120 brings several Foundation priorities to bear,” said Deborah W. Brooks, MJFF president and CEO. “These include investigating the neurorestorative properties of neurotrophic factors, advancing translational research, and shortening the time it takes to turn basic research advances into meaningful therapies for patients.” She added that without Foundation support, the trial would proceed at a slower pace, with fewer clinical assessments and less imaging — resulting in longer timelines to completion.

“We’re pleased to have Fox Foundation support to optimize our clinical tests of CERE-120,” said Jeffrey M. Ostrove, Ph.D., president and CEO of Ceregene. “This funding will allow us to gather, in the shortest time possible, the data needed to know if we are on to a safe therapy that might slow or stop the progression of the disease — something no treatment on today’s market can do.” 

Neurotrophic factors, also known as growth factors, have long been considered one of the most promising avenues for Parkinson’s therapies, as 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, and the two have similar pharmacological properties: Both maintain survival of the dopamine-producing nerve cells that are required for normal bodily movement and that degenerate in people with Parkinson’s.

“Extensive studies in animal models, including the most widely accepted models of Parkinson’s disease, have consistently demonstrated that CERE-120 is safe and well tolerated in animals even at doses hundreds of times higher than the equivalent doses being tested in humans. These studies also demonstrate that CERE-120 may be able to improve symptoms as well as slow the progression of Parkinson’s disease,” stated Raymond T. Bartus, Ph.D., Ceregene’s COO and principal investigator on the grant.

The MJFF funding supplements Ceregene’s own multi-million dollar investment in the study, which is under way at the University of California, San FranciscoMedicalCenter and RushUniversityMedicalCenter in Chicago. Other MJFF investments in neurotrophic factors to date include a current LEAPS (Linked Efforts to Accelerate Parkinson’s Solutions) project investigating encapsulated cell technology as a delivery mechanism for GDNF and two Community Fast Track 2004 projects: One evaluated two proteins from the neuregulin family of growth factors as a potential therapeutic agents in animal models, and another examined a molecule called pleiotrophin as a potential neuroprotective/restorative agent for dopamine neurons in animal models.

The Ceregene grant was funded in its entirety by the Pioneer Fund, a private family foundation that supports endeavors including medical research, under the Foundation’s Clinical Discovery program. Clinical Discovery is designed to stimulate well-designed clinical research projects focused on potentially high-impact approaches to Parkinson’s disease. “We committed to funding the CERE-120 trial because of its great potential benefit for people with Parkinson’s,” said Scott Hamilton, Olympic gold medalist and Pioneer Fund Board member. “We are pleased to work together with the Fox Foundation to drive the kind of cutting-edge science that can lead to meaningful advances for the millions touched by this disease.”

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