SAN DIEGO, Calif., April 25 /PRNewswire/ -- Ceregene, Inc. scientific co-
founder, Mark Tuszynski M.D., Ph.D., published today in the May 2005 online
edition of Nature Medicine the results of the first ever Phase I clinical
study for the treatment of 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
"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 encouraging and strongly support our CERE-110 clinical
program using an AAV2-based vector to deliver NGF to Alzheimer's patients."
Ceregene is currently sponsoring a Phase I study of CERE-110, an off-the-self
version that uses direct NGF gene delivery to the brain. This clinical study
is currently underway at Rush University Medical Center in Chicago.
"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
and the study's principal investigator. "This would also represent the first
therapy for a human neurological disease that functions by preventing cell
The Phase I trial, sponsored by 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.
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. The six patients who
safely completed the NGF delivery procedure were evaluated using quantitative
measurements of the Mini Mental Status Examination (MMSE) and the Alzheimer's
Disease Assessment Scale-Cognitive Subscale (ADAS-Cog). 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. Gene therapy with NGF
was well tolerated in this trial. Two patients early in the study experienced
hemorrhage during the neurosurgical procedure which has been modified and has
eliminated this problem to date.
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.
Contact: Jeffrey M. Ostrove, Ph.D.
President and Chief Executive Officer
SOURCE Ceregene, Inc.