Thursday April 29, 7:07 am ET Encouraging Results Reported in Initial Phase I Clinical Trial SAN DIEGO, April 29 /PRNewswire/ —
Ceregene, Inc., today announced that based on preliminary findings in a study conducted at the University of California, San Diego (UCSD), it will advance its gene therapy for Alzheimer's disease into further clinical development. A report this week of an initial Phase I clinical trial of Alzheimer's disease gene therapy provided early indications of a potential reduction in the advancement of the disease in patients with mild to moderate Alzheimer's disease. In addition, it was also observed by radiologic studies that the brain metabolic activity increased in the treated patients in comparison to non treated Alzheimer's disease patients.
These data were presented Tuesday at the American Academy of Neurology Meeting in San Francisco by Mark Tuszynski, M.D., Ph.D., professor of neurosciences at the UCSD School of Medicine, and principal investigator on the Alzheimer's disease gene therapy study. Ceregene has exclusive worldwide rights to the technology used in the Phase I study.
"We are encouraged by the data presented this week and plan to advance this novel treatment strategy into additional clinical trials including a Phase I/II Alzheimer's gene therapy study expected to begin in the near future," stated Jeffrey M. Ostrove, Ph.D., president and chief operating officer of Ceregene. "Alzheimer's disease is a significant unmet medical need affecting more than four million people in the United States, and to date, no treatments to prevent progression or reverse Alzheimer's symptoms have been developed, so we are excited about the possibility of developing a product to help these patients in need." "These results are intriguing," stated Dr. Tuszynski. "If these effects are borne out in larger, controlled trials, this could be a significant advance over existing therapies for Alzheimer's disease."
The University of California holds the patent to the technology employed in the initial clinical trial, and the exclusive worldwide licensee of the technology is Ceregene, Inc. 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. The autopsy of a patient on the study who died of a heart attack five weeks after the surgical procedure, showed active NGF production in the brain and a growth response of brain cells to the NGF delivery.
The procedure initially was performed while patients were awake but lightly sedated. Two patients moved as the cells were being injected, resulting in bleeding in the brain. Following these events, the protocol was redesigned with patients given general anesthesia during the procedure, and subsequent procedures were performed without complication. Based on these results, Dr. Tuszynski concludes that the NGF implants, when performed with patients fully anesthetized, appear to be safe and well tolerated by patients.
The Phase I uncontrolled trial was conducted in eight patients with early-stage Alzheimer's disease. Cells from the patients' skin were genetically modified to produce nerve growth factor (NGF), a naturally occurring protein that maintains survival of nerve cells in the brain, and were then surgically implanted into the nucleus basalis of Meynert, 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.
Ceregene is planning to initiate additional gene therapy clinical trials during the coming year, including trials in Alzheimer's disease (CERE-110) and Parkinson's disease (CERE-120) expected to begin during 2004, and a trial in amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) (CERE-130) expected to be initiated during 2005. The Phase I/II study in Alzheimer's disease gene therapy is expected to begin in the near future and will evaluate the delivery to the brain of a non patient-specific, "off-the-shelf" product which is comprised of an adeno-associated viral (AAV) gene delivery system carrying the NGF gene.
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 majority-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 product pipelines, the relevance of intellectual property, and clinical and preclinical programs including those associated with Alzheimer's disease gene therapy 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 which 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. The University of California and some of its investigators have a financial interest in Ceregene.