Press Releases
04/10/01
CEREGENE CONFIRMS TREATMENT OF FIRST PATIENT IN PHASE 1 STUDY OF ALZHEIMER'S DISEASE GENE THERAPY
First Patient Ever Treated in Human Gene Therapy Study for Alzheimer's Disease.
SAN DIEGO, Calif., April 10, 2001 - Ceregene, Inc. confirmed today that the first patient has been treated in a Phase 1 clinical study for Alzheimer's disease gene therapy at the University of California, San Diego (UCSD) School of Medicine. Ceregene, Inc., a majority-owned subsidiary of Cell Genesys, Inc. (Nasdaq: CEGE) is the exclusive licensee of the technology and product rights for this therapy.
"Ceregene is excited by this groundbreaking study involving the first ever gene therapy trial for Alzheimer's disease," said Jeffrey M. Ostrove, Ph.D., president and chief operating officer of Ceregene. "Several successful preclinical gene therapy studies for both Alzheimer's disease and Parkinson's disease have demonstrated the potential of Ceregene's gene therapy technologies, and we are pleased that human clinical studies are now under way."
The Phase 1 trial, sponsored by UCSD and led by UCSD neurologist, Mark Tuszynski, M.D., Ph.D., involves surgical implantation of cells producing nerve growth factor (NGF), a naturally occurring protein that maintains survival of nerve cells in the brain. The primary goal of this study is to determine that the gene therapy procedure is safe. The secondary goal is to determine whether NGF produced by the cells implanted into the brain can prevent the death of some nerve cells that are affected by Alzheimer's disease and whether it can enhance the function of certain 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, a common neurosurgical procedure, into the region of the brain called the nucleus basalis of MeynertUan area of the brain known to undergo serious degeneration in Alzheimer's disease. The patient will be closely monitored for a year and then evaluated annually for an indefinite period. Eight patients are expected to be enrolled into the Phase 1 study. In the future, Ceregene plans to conduct in vivo (genetic modification inside the body) studies for both Alzheimer's disease and Parkinson's disease. This human clinical study follows a number of encouraging animal studies conducted by Dr. Tuszynski and colleagues previously reported in the scientific literature. In a September issue of the journal, Proceedings of the National Academy of Sciences (PNAS), Dr. Tuszynski's team reported that monkeys exhibited atrophy of 40 percent of cholinergic neurons due to the normal aging process; these atrophic changes were reversed upon administration of NGF by this procedure. In a separate PNAS article in February 2001, these scientists demonstrated that the delivery of the NGF gene to monkeys restored normal levels and activity of axons (message transmitters between neurons), after observing that quantities of axons in aged monkeys had significantly declined compared with younger monkeys.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development and commercialization of gene therapies for neurologic disorders. Ceregene was launched in January 2001 as a majority-owned subsidiary of Cell Genesys, Inc. through the acquisition of Neurologic Gene Therapeutics, a private start-up company based in San Diego. Cell Genesys contributed $10 million to Ceregene, together with access to technology and patents in the central nervous system (CNS) gene therapy area, in exchange for approximately 60 percent ownership of the new company. Ceregene has exclusively licensed technology related to the Phase 1 human clinical trial for Alzheimer's disease currently under way. In addition, Ceregene is conducting advanced preclinical studies in Parkinson's disease. These programs were pioneered by the founders of Neurologic Gene Therapeutics prior to the formation of Ceregene.