I know it doesn't mention MS but I think it's very encouraging that gene therapy is helping a neurdegenerative disease like Parkinson's. It could help us one day too.
Ceregene Presents Long Term Follow-Up Data From Phase 1 Trial of CERE-120 Demonstrating Improved Motor Function in Parkinson's Patients
April 30th, 2007
(NewsRx.com) -- Ceregene, Inc., a biopharmaceutical company, today presented long term follow-up data from a Phase 1 clinical trial of CERE-120, a gene therapy product in development for the treatment of Parkinson's disease, demonstrating a 36 percent (p<0.001) reduction in Parkinson's symptoms at 12 months after administration, as measured by the Unified Parkinson's Disease Rating Scale (UPDRS) motor "off" score ("motor off" meaning patients were off Parkinson's medication at evaluation time). CERE-120 was also shown to be well tolerated in this study. The 12-month results were presented today by Philip Starr, M.D., Ph.D., associate professor of neurosurgery at the University of California, San Francisco at the American Association of Neurological Surgeons (AANS) annual meeting in Washington D.C. These data were also highlighted by the AANS in a specific CERE-120 press release issued by the Association.
The Phase 1 trial was an open-label study conducted in 12 patients with advanced Parkinson's disease at two clinical trial sites -- University of California, San Francisco and Rush University Medical Center in Chicago. All 12 patients enrolled in the study underwent stereotactic neurosurgery to deliver CERE-120 into their putamen -- a region of the brain affected by the degeneration of neurons in Parkinson's disease.
CERE-120 was delivered at two different doses, and patients receiving the low dose demonstrated approximately 36 percent improvement in UPDRS motor "off" scores by nine months and patients receiving the four-fold higher dose showed a similar effect three months sooner. This statistically significant, 36 percent improvement was maintained in both groups at the 12-month mark, the final follow-up time point in the study. Patients also demonstrated a 50 percent reduction in hours of "off" time (i.e., time when normal Parkinson's medication was ineffective and symptoms were troubling to the patient) and a doubling of good quality "on" time without dyskinesias (i.e., time when a patient is functioning well) according to self-reported diaries.
This Phase 1 clinical trial was partially supported by a grant from The Michael J. Fox Foundation for Parkinson's Research. Based on the results of the Phase 1 study, the Foundation has provided a $1.9 million grant, which will provide partial funding for Ceregene's ongoing Phase 2 trial.
"We are pleased with the results of this early study which suggests that the majority of patients treated with CERE-120 may have exhibited significant and stable improvement for a full year after receiving a single administration of CERE-120," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "Each of our products, much like CERE-120, is aimed at unmet neurodegenerative diseases including Parkinson's disease, Alzheimer's disease and Amyotrophic Lateral Sclerosis (AML or Lou Gehrig's disease) with the goal of not only improving the symptoms but also preventing progression of the disease."
"Through stereotactic surgery, we are able to administer CERE-120 in a highly targeted fashion to one area of the brain that Parkinson's disease affects, and we were able to do this safely," stated Dr. Starr.
"Given the encouraging data from the Phase 1 trial of CERE-120 in Parkinson's disease, we are conducting a follow-on Phase 2 clinical trial that is currently enrolling patients at nine clinical trial sites in the United States," said Raymond T. Bartus, Ph.D., Ceregene's senior vice president of clinical and preclinical R&D and chief operating officer. "The data from the Phase 1 trial are reflective of the impressive results we gathered from preclinical studies, which demonstrated the ability of CERE-120 to stimulate the survival and improve the function of key neuronal cells affected by Parkinson's disease, as well as an excellent safety profile over a wide range of CERE-120 dose levels."
Keywords: Biotechnology, Central Nervous System Disease, Clinical Trial Research, Gene Therapy, Genetics, Genomics, Parkinson Disease, Ceregene Inc.
This article was prepared by NewsRx editors from staff and other reports. Copyright 2007, NewsRx.com.