SAN DIEGO, Calif., May 2 /PRNewswire/ -- Ceregene, Inc. today presented
interim clinical data from a Phase 1 trial of CERE-110, a gene therapy
product designed to deliver nerve growth factor (NGF) for the treatment of
Alzheimer's disease. After one year of follow-up, the results for the six
patients who received CERE-110 in the trial indicate that a single
administration of the therapy was well tolerated, and an interim analysis
suggested that the therapy had the potential to reduce the rate of
cognitive decline and increase brain metabolism. These data were presented
today by Zoe Arvanitakis, M.D., and David Bennett, M.D., co-principal
investigators, Rush Alzheimer's Disease Center, Dept. of Neurological
Sciences, Rush University Medical Center, at the American Academy of
Neurology Meeting in Boston, Massachusetts.
The Phase 1 trial at Rush University Medical Center was a single-site,
open-label study involving six patients with mild-to-moderate Alzheimer's
disease and was conducted for the purpose of evaluating whether
CERE-110-NGF administered by an adeno-associated viral gene delivery
system-could be safely delivered. All six patients enrolled in the study
underwent stereotactic neurosurgery to deliver CERE-110 into a specific
region of the brain called the Nucleus Basalis of Meynert (NBM) -- an area
that is a source for the production of cholinergic neurons, which are known
to undergo profound degeneration in patients with Alzheimer's disease. The
administration of CERE-110 was generally well tolerated.
The six patients underwent cognitive testing, and the data suggest that
a reduction in the rate of cognitive decline may have been achieved. PET
(positron emission tomography) scans were also obtained of the patients'
brains at baseline, six and 12 months, and increases in brain metabolism
were observed in several cortical regions at six months (p<0.05). Further
increases in metabolism were measured at 12 months (p<0.05), representing a
potential reversal of patterns typically observed in Alzheimer's disease.
"Based on the encouraging data from this Phase 1 trial of CERE-110, we
are planning a larger, controlled, Phase 2 trial that we expect to start in
2008. We are currently discussing potential collaborations for this program
for the co-development of this promising therapy, CERE-110 for Alzheimer's
disease," stated Jeffrey M. Ostrove, Ph.D., president and chief executive
officer of Ceregene. "Similar to the other therapies in our pipeline which
target Parkinson's disease and Lou Gehrig's disease, CERE-110 may have the
ability to not only treat the symptoms of Alzheimer's disease but to also
treat the underlying disease potentially resulting in slowed disease
progression."
"Although this initial study is ongoing and we will need to wait until
its completion for the final results, we believe that, to date, there are
no serious safety issues with the gene expression associated with this
experimental drug in our six patients with Alzheimer's disease," stated Dr.
Arvanitakis.
"CERE-110 may have the potential to substantially reduce the loss of
the key neuronal cells affected in patients with Alzheimer's disease.
Should the clinical development of this therapy be successful, CERE-110
would offer the possibility of delaying the course of Alzheimer's disease,
a real improvement over existing therapies," stated Mark Tuszynski, M.D.,
Ph.D., professor of neurosciences at the University of California, San
Diego School of Medicine and principal investigator of an earlier Phase 1
trial of Nerve Growth Factor gene transfer in Alzheimer's Disease.
"These data add to the accumulating evidence from our studies in both
humans and animals that neurotrophic factors can be delivered safely and
effectively using gene transfer, thus providing the potential to restore
function and slow further degeneration in many different neurodegenerative
diseases," added Raymond T. Bartus, Ph.D., Ceregene's senior vice president
of clinical and preclinical R&D and chief operating officer. "These results
in Alzheimer's patients, wherein we target degenerating cholinergic
neurons, builds upon on the research of the past 30 years, showing that
improving the function of these neurons can significantly reduce the memory
loss in this disease".
About CERE-110
CERE-110 is composed of an AAV vector carrying the gene for NGF, a
naturally occurring protein that maintains survival of nerve cells in the
brain. CERE-110 is surgically implanted by stereotactic injection into the
NBM, 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. Delivery of NGF using an AAV vector may have the potential
to induce sustained expression of NGF, resulting in the restoration of
normal functioning of the key neuronal cells affected in Alzheimer's
disease patients.
About Alzheimer's Disease
Alzheimer's disease is a progressive disorder of the brain that
gradually affects one's memory and ability to learn, reason, communicate
and carry out daily activities. There are now more than five million people
in the United States living with Alzheimer's disease, and there is
currently no cure.
About Ceregene
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,
and with CERE-120 for Parkinson's disease. CERE-130 is in late preclinical
development for ALS. Ceregene was launched in January 2001 and is a former
subsidiary of Cell Genesys, Inc. (Nasdaq: CEGE), which is headquartered in
South San Francisco, CA. Ceregene's investors include Alta Partners, MPM
Capital, Investor Growth Capital and Cell Genesys, as well as Hamilton
BioVentures and, California Technology Partners.
Contact:
Jeffrey M. Ostrove, Ph.D.
President and CEO
Ceregene, Inc.
(858) 458-8808
SOURCE Ceregene, Inc.
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Related links:
http://www.cellgenesys.com/
CONTACT:
Jeffrey M. Ostrove, Ph.D., President and CEO,
Ceregene, Inc., +1-858-458-8808
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