Biotechnology company Ceregene, Inc.has completed enrollment in its double-blind phase II clinical study of CERE-110 (AAV-NGF), a gene therapy product designed to deliver nerve growth factor (NGF) for the treatment of Alzheimer's disease. The clinical study was carried out in collaboration with the Alzheimer's Disease Cooperative Study (ADCS) based at the University of California San Diego (UCSD) and funded by a grant from the National Institute on Aging (NIA), part of the National Institutes of Health (NIH).
Forty-nine (49) patients with mild to moderate Alzheimer's disease were treated with CERE-110 at 10 clinical sites throughout the US. Approximately half of the patients received CERE-110 while the other half received an appropriate sham (placebo) surgery control treatment. Patients will be followed for a minimum of two years with respect to safety, brain imaging, as well as measures of cognitive function and quality of life. Standard tests used in Alzheimer's clinical trials.
"We are very excited that enrollment in the first blinded study of NGF gene therapy for Alzheimer's disease is now completed and appreciate the courage of all of the patients and their family members that enrolled in this very important study. We also appreciate the efforts of the ADCS and the National Institute on Aging for carrying out and supporting the operational aspects of this study" stated Jeffrey M Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc., the developer of CERE-110. Dr Ostrove added, "In addition to CERE-110 for Alzheimer's disease, we have also completed enrollment in two controlled phase II clinical trials of CERE-120 for Parkinson's disease. At this time, we would like to recognize the leadership role of Raymond T Bartus, Ph.D., our chief scientific officer who led the development efforts for both of these potential products for the treatment of our most common neurodegenerative disorders.”
"We were very pleased to be involved in evaluating CERE-110. We believe CERE-110 may prove to be an effective therapy for Alzheimer's disease," stated Paul Aisen, MD, project director of the phase II trial and director of the Alzheimer's Disease Cooperative Study at University of California San Diego, a preeminent research consortium for testing new treatments for Alzheimer's disease. "If the results prove positive, which is our hope, this therapy could help the over five million people in the US currently living with Alzheimer's disease in need of more effective treatment options."
CERE-110 is composed of an adeno-associated viral (AAV) vector carrying the gene for NGF, a naturally occurring protein that maintains survival of nerve cells in the brain. CERE-110 is surgically injected into the Nucleus Basalis of Meynert (NBM), a brain region where cholinergic cell degeneration occurs in Alzheimer's disease. The cholinergic system is important in memory and cognitive function, and a restoration in the function of this system may improve memory in individuals with Alzheimer's disease. Delivery of NGF using an AAV vector should have the potential to induce sustained expression of NGF, resulting in long-lasting restoration of the function.
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 focused on the delivery of nervous system growth factors for the treatment of neurodegenerative disorders using gene delivery.