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news.Ceregene has reported data showing that CERE-120, a gene therapy product in development for the treatment of Parkinson's disease, was well tolerated and appeared to reduce symptoms by approximately 40%.
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The study was authored by UCSF and Rush University Medical Center.
CERE-120 carries the gene for neurturin, a naturally occurring protein, whose role is to keep dopamine-secreting neurons alive and functioning normally. All patients enrolled in the study underwent neurosurgery to deposit CERE-120 into their putamen. The putamen is a region of the brain that undergoes degeneration and reduced dopamine production in Parkinson's disease patients.
“Targeted delivery of the trophic factor neurturin is a compelling approach to treating Parkinson's disease,” said. Dr William Marks, principal investigator of the study. “The safety data and preliminary efficacy data that we have seen in this phase I study are encouraging. Clearly, a larger-scale study is warranted.”
“The development of growth factors as a treatment for neurodegenerative diseases has been hampered by the difficulty of delivering them specifically to the targeted areas that need their neuroprotective properties. We believe our programs increasingly demonstrate that gene transfer may represent a safe and effective means of solving this age-old problem,” noted Raymond Bartus, Ceregene's COO.
The study was supported in part by a grant from The Michael J. Fox Foundation for Parkinson's research. Based on the initial results, the foundation also revealed plans to partially fund a phase II study with a $1.9 million grant.