SAN DIEGO, Calif., May 02, 2007 /PRNewswire/ -- Ceregene, Inc. today presented interim clinical data from a Phase 1 trial of CERE-110, a gene therapy product designed to deliver nerve growth factor (NGF) for the treatment of Alzheimer's disease. After one year of follow-up, the results for the six patients who received CERE-110 in the trial indicate that a single administration of the therapy was well tolerated, and an interim analysis suggested that the therapy had the potential to reduce the rate of cognitive decline and increase brain metabolism. These data were presented today by Zoe Arvanitakis, M.D., and David Bennett, M.D., co-principal investigators, Rush Alzheimer's Disease Center, Dept. of Neurological Sciences, Rush University Medical Center, at the American Academy of Neurology Meeting in Boston, Massachusetts.
The Phase 1 trial at Rush University Medical Center was a single-site, open-label study involving six patients with mild-to-moderate Alzheimer's disease and was conducted for the purpose of evaluating whether CERE-110-NGF administered by an adeno-associated viral gene delivery system-could be safely delivered. All six patients enrolled in the study underwent stereotactic neurosurgery to deliver CERE-110 into a specific region of the brain called the Nucleus Basalis of Meynert (NBM) -- an area that is a source for the production of cholinergic neurons, which are known to undergo profound degeneration in patients with Alzheimer's disease. The administration of CERE-110 was generally well tolerated.
The six patients underwent cognitive testing, and the data suggest that a reduction in the rate of cognitive decline may have been achieved. PET (positron emission tomography) scans were also obtained of the patients' brains at baseline, six and 12 months, and increases in brain metabolism were observed in several cortical regions at six months (p<0.05). Further increases in metabolism were measured at 12 months (p<0.05), representing a potential reversal of patterns typically observed in Alzheimer's disease.
"Based on the encouraging data from this Phase 1 trial of CERE-110, we are planning a larger, controlled, Phase 2 trial that we expect to start in 2008. We are currently discussing potential collaborations for this program for the co-development of this promising therapy, CERE-110 for Alzheimer's disease," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "Similar to the other therapies in our pipeline which target Parkinson's disease and Lou Gehrig's disease, CERE-110 may have the ability to not only treat the symptoms of Alzheimer's disease but to also treat the underlying disease potentially resulting in slowed disease progression."
"Although this initial study is ongoing and we will need to wait until its completion for the final results, we believe that, to date, there are no serious safety issues with the gene expression associated with this experimental drug in our six patients with Alzheimer's disease," stated Dr. Arvanitakis.
"CERE-110 may have the potential to substantially reduce the loss of the key neuronal cells affected in patients with Alzheimer's disease. Should the clinical development of this therapy be successful, CERE-110 would offer the possibility of delaying the course of Alzheimer's disease, a real improvement over existing therapies," stated Mark Tuszynski, M.D., Ph.D., professor of neurosciences at the University of California, San Diego School of Medicine and principal investigator of an earlier Phase 1 trial of Nerve Growth Factor gene transfer in Alzheimer's Disease.
"These data add to the accumulating evidence from our studies in both humans and animals that neurotrophic factors can be delivered safely and effectively using gene transfer, thus providing the potential to restore function and slow further degeneration in many different neurodegenerative diseases," added Raymond T. Bartus, Ph.D., Ceregene's senior vice president of clinical and preclinical R&D and chief operating officer. "These results in Alzheimer's patients, wherein we target degenerating cholinergic neurons, builds upon on the research of the past 30 years, showing that improving the function of these neurons can significantly reduce the memory loss in this disease".
CERE-110 is composed of an AAV vector carrying the gene for NGF, a naturally occurring protein that maintains survival of nerve cells in the brain. CERE-110 is surgically implanted by stereotactic injection into the NBM, a deep brain region where cholinergic cell degeneration occurs in Alzheimer's disease. The cholinergic system is important in memory and cognitive function, and a reversal in the blockade of this system may restore memory. Delivery of NGF using an AAV vector may have the potential to induce sustained expression of NGF, resulting in the restoration of normal functioning of the key neuronal cells affected in Alzheimer's disease patients.
About Alzheimer's Disease
Alzheimer's disease is a progressive disorder of the brain that gradually affects one's memory and ability to learn, reason, communicate and carry out daily activities. There are now more than five million people in the United States living with Alzheimer's disease, and there is currently no cure.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development of gene therapies for neurodegenerative disorders. Ceregene is in the clinic with CERE-110, an AAV2 based vector expressing nerve growth factor that is being tested as a treatment for Alzheimer's disease, and with CERE-120 for Parkinson's disease. CERE-130 is in late preclinical development for ALS. Ceregene was launched in January 2001 and is a former subsidiary of Cell Genesys, Inc. , which is headquartered in South San Francisco, CA. Ceregene's investors include Alta Partners, MPM Capital, Investor Growth Capital and Cell Genesys, as well as Hamilton BioVentures and, California Technology Partners.
Contact: Jeffrey M. Ostrove, Ph.D. President and CEO Ceregene, Inc. (858) 458-8808
CONTACT: Jeffrey M. Ostrove, Ph.D., President and CEO, Ceregene, Inc.,+1-858-458-8808
Web site: http://www.cellgenesys.com//
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