Ceregene Scientific Co-Founder Publishes Alzheimer’s Disease Clinical Trial Results In Nature Medici
Promising Phase I Data Demonstrated in Gene Therapy Study for Alzheimer’s Disease
SAN DIEGO, Calif., April 30, 2005—Ceregene, Inc. scientific co-founder, Mark Tuszynski M.D., Ph.D., published today in the May 2005 edition of Nature Medicine the results of the first ever Phase I clinical study for Alzheimer’s disease using gene therapy. Ceregene, Inc. has licensed the technology and product rights used in this program and have utilized the data in its current Alzheimer’s gene therapy clinical studies.
“Ceregene is excited to be involved in such a promising breakthrough approach delivering growth factors to treat neurodegenerative diseases,” stated Jeffrey, M. Ostrove, Ph.D., president and chief executive officer of Ceregene. “The results of this Phase I study of nerve growth factor (NGF) in Alzheimer’s disease are extremely encouraging and strongly support our CERE-110 clinical program using an AAV2-based vector to deliver NGF to Alzheimer’s patients.”
“If validated in further clinical trials, this would represent a substantially more effective therapy than current treatments for Alzheimer’s disease,” said Mark Tuszynski, M.D., Ph.D., UCSD professor of neurosciences, neurologist with the VA San Diego Healthcare System, and the study’s principal investigator. “This would also represent the first therapy for a human neurological disease that functions by preventing cell death.”
The Phase I trial, sponsored by UCSD neurologist Mark Tuszynski, M.D., Ph.D., involved surgical implantation of cells producing nerve growth factor (NGF), a natural brain survival molecule. The primary goal was to determine that the gene therapy procedure is safe. The secondary goal was to determine whether NGF produced by the cells implanted into the brain could prevent the death of some nerve cells that are affected in Alzheimer’s disease and whether it can enhance the function of some remaining brain cells.
During the study, the cells, which originally are extracted from the patient’s skin, are genetically modified to express NGF. The cells are then administered by stereotactic injection through five implants into the region of the brain called the nucleus basalis of Meynert—an area of the brain known to undergo serious degeneration in Alzheimer’s disease.
In the Nature Medicine published report, cognitive measurements were assessed pretreatment, at the time of treatment and at regular intervals following administration of the NGF producing cells. Quantitative measurements of the Mini Mental Status Examination (MMSE) and the Alzheimer’s Disease Assessment Scale-Cognitive Subscale, or ADAS-Cog in the six patients who safely completed the NGF delivery procedure were evaluated. Over an average post-treatment period of 22 months, the rate of decline on the MMSE was reduced by as much as 51 percent. The ADAS-Cog also showed improvements in rates of decline that paralleled the MMSE findings.
Using PET brain-imaging studies, scans in four subjects showed significant increases in the brain’s metabolic activity as measured by absorption of a radioisotope. This increase was observed in most cortical regions that receive cholinergic input from the treated area of the brain called the nucleus basalis and in the cerebellum, a structure associated with cortical plasticity.
Ceregene is currently sponsoring a Phase I study of CERE-110, an off-the-self version of Mark Tuszynski’s product that uses direct NGF gene delivery to the brain and thereby eliminates the need for grafting cells. The clinical study is currently underway at Rush University Medical Center in Chicago. Ceregene holds an exclusive license from Genentech for the use of NGF in these gene therapy delivery systems. In addition to this CERE-110 study for Alzheimer’s disease, Ceregene has developed CERE-120, an AAV2-based vector to deliver Neurturin, another growth factor that is related to GDNF which has been reported to function in the treatment of Parkinson’s disease. CERE-120 has been reviewed by the Food and Drug Administration and has an effective IND as well as approval from the NIH Recombinant DNA Advisory Committee (RAC). Clinical trials are expected to start in the near future.
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. CERE-120 for Parkinson’s disease will be tested in the clinic shortly and CERE-130 is in late preclinical development for ALS. Ceregene was launched in January 2001 and is a minority-owned subsidiary of Cell Genesys, Inc. (Nasdaq: CEGE), which is headquartered in South San Francisco, CA. Ceregene closed a $32M Series B financing in August 2004 that was co-led by Alta Partners and MPM Capital. For more information, visit the Ceregene web site. http://www.ceregene.com/
5/4/2005
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