Ceregene Begins Phase I Clinical Trial of Novel Gene Therapy Product for Patients With Alzheimer's Disease
Tuesday September 21, 7:00 am ET
It's early, Starbucks hasn't kicked in yet, someone please explain "off the shelf", sounds spooky to me, mixing genes
First Clinical Trial to Test Off-the-Shelf Gene Therapy Product
SAN DIEGO, Sept. 21 /PRNewswire-FirstCall/ -- Ceregene, Inc., a biotechnology company focused on gene therapies for neurodegenerative disorders, today confirmed that the first patient has been treated in a Phase I clinical study of CERE-110, a novel gene therapy product that involves in vivo delivery of nerve growth factor (NGF) genes through an adeno-associated viral (AAV) vector delivery system. The naturally occurring NGF gene encodes the NGF protein that maintains survival of nerve cells in the brain that are required for memory and cognitive function. This clinical trial is the first to test the company's growth factor gene therapy delivery system in a non patient-specific, "off-the-shelf" product formulation for patients with Alzheimer's disease. Ceregene owns exclusive technology and product rights to this therapy.
"We are pleased to have initiated this groundbreaking study involving the first ever off-the-shelf gene therapy product for Alzheimer's disease," said Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "Several successful preclinical studies have demonstrated the potential of Ceregene's in vivo gene therapy technologies and a pilot human study of ex vivo gene therapy for Alzheimer's disease was recently reported with encouraging results. We are pleased that human clinical studies of our off-the-shelf product format are now under way."
This study is being led by David Bennett, M.D., neurologist and director of the Rush University Alzheimer's Disease Center, and Zoe Arvanitakis, M.D., neurologist at Rush University Medical Center in Chicago. Dr. Roy Bakay, a neurosurgeon at Rush University Medical Center, performs the surgery.
This new Phase I study involves in vivo (inside the body) gene therapy delivery of NGF. Ceregene's in vivo gene therapies utilize a non-replicating viral vector to deliver therapeutic genes to the nervous system, where they express proteins with the ability to prevent neurodegeneration and restore normal nerve function. The adeno-associated viral (AAV) vector, which has never been causally associated with disease in humans, is an important gene delivery tool that has demonstrated ability to efficiently and stably express genetic information in non-dividing target cells, such as the neurons degenerating in Alzheimer's disease, Parkinson's disease and ALS and to achieve potentially long-term, therapeutic benefit. The goal of this study is to determine the safety and efficacy of this new gene therapy system. Efficacy will be measured by memory and cognitive tests, as well as brain imaging studies.
"Today, Alzheimer's disease represents a major unmet medical need, and current therapies are limited to drugs that attempt to increase the levels of certain chemical messengers in the brain," stated Raymond T. Bartus, Ph.D., Ceregene's vice president for research and development. "This NGF gene therapy strategy is attempting to stop the underlying cause of the disease by reversing the deterioration of nerves referred to as cholinergic neurons."
In April 2004, encouraging results from a previous Phase I study of NGF gene therapy in mild to moderate Alzheimer's disease were presented at the American Academy of Neurology Meeting by Mark Tuszynski, M.D., Ph.D., professor of neurosciences at UCSD and cofounder of Ceregene and consultant in the current study. This initial Phase I study involved ex vivo (outside the body) genetic modification of cells to express NGF. Cells from the patients' skin were genetically modified to produce NGF and were then surgically implanted into the brain. In the six evaluable, early-stage Alzheimer's disease patients, an approximately 40-50 percent reduction was observed in their annual rate of decline on the measured cognitive function scales compared to their pre-operative function. The subjects showed a reduced rate of decline that persisted throughout the 1.5 to two-year period of the study. With currently available pharmacologic therapy, the average rate of decline in cognitive function is up to six percent with a median duration of three to six months. Additionally, Positron Emission Tomography (PET) scans of the patients' brains showed increased metabolic activity in the areas of the brains of patients following treatment with NGF, compared with non treated Alzheimer's disease patients.
Ceregene has an active clinical program in Alzheimer's disease (CERE-110) and is planning to initiate several additional clinical trials during the coming year, including trials in Parkinson's disease (CERE-120) and amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease (CERE-130) which is expected to be initiated during 2005.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development and commercialization of gene therapies for neurological disorders including Alzheimer's disease, Parkinson's disease and amyotrophic lateral sclerosis (ALS). Ceregene was launched in January 2001 and is a minority-owned subsidiary of Cell Genesys, Inc. (Nasdaq: CEGE - News), which is headquartered in South San Francisco, CA.
Statements made herein about Ceregene and Cell Genesys, other than statements of historical fact, including statements about clinical programs are forward-looking statements and are subject to a number of uncertainties that could cause actual results to differ materially from the statements made including risks associated with the success of research and development programs, clinical trials, the regulatory approval process for clinical trials, competitive technologies and products, patents, continuation of option agreements and the need for additional financings. For information about these and other risks that may affect Ceregene and Cell Genesys, please see the Cell Genesys Annual Report on Form 10-K dated March 4, 2004 as well as Cell Genesys' reports on Form 10-Q and 8-K and other reports filed from time to time with the Securities and Exchange Commission.
CONTACT: Jeffrey M. Ostrove, Ph.D., President and Chief Executive Officer of Ceregene, +1-858-458-8800.
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