Sangamo BioSciences, Inc.
SGMO announced today the presentation of positive data from the
Phase 1 clinical trial of CERE-110 (AAV-NGF), a gene therapy approach designed
to deliver nerve growth factor (NGF) for the treatment of Alzheimer's disease
(AD). This novel product was developed by Ceregene, Inc., which was recently
acquired by Sangamo. The data were presented at the Sixth Clinical Trials on
Alzheimer's Disease (CTAD) Meeting, which is being held in San Diego from
November 14-16, 2013.
(Logo: http://photos.prnewswire.com/prnh/20130102/SF35903LOGO)
The data from this dose escalation study demonstrate that surgical delivery of
CERE-110 to the brain results in the long-term expression of bioactive NGF,
the therapeutic protein. Clinicians also observed apparent stabilization of
brain cell metabolic activity in treated subjects, as determined by PET-scans
measuring glucose use, which may reflect a slowing of cell deterioration. The
treatment was well-tolerated at all dose levels.
"These early clinical data demonstrate that this therapeutic approach is
feasible, well-tolerated and results in appropriate delivery of the
therapeutic, NGF protein, to the intended target cells in the brain," said
Paul Aisen, M.D., Project Director of the ongoing Phase 2 clinical trial of
CERE-110 and director of the Alzheimer's Disease Cooperative Study (ADCS) at
the University of California San Diego, a preeminent research consortium for
testing new treatments for Alzheimer's disease. "We are very pleased to be
involved in the Phase 2 clinical trial, which is a placebo-controlled study,
and will enable further evaluation of the efficacy of CERE-110. Alzheimer's
disease has few effective treatment options, and this therapy could help the
over five million people in the U.S. currently living with the disease."
The Phase 2 clinical study of CERE-110 is being carried out in collaboration
with the ADCS and is funded by a grant from the National Institute on Aging
(NIA), part of the National Institutes of Health (NIH).
"We are very encouraged by the Phase 1 data from the CERE-110 study," stated
Edward Lanphier, Sangamo's president and CEO. "These positive data supported
the further testing of this novel approach in a Phase 2 clinical trial to
evaluate efficacy. The Phase 2 study is fully-funded and fully-enrolled and
currently in the two year follow-up phase. We look forward to presenting data
from the clinical trial in 2015."
The data, which have been accepted for publication in Alzheimer's and
Dementia, the official publication of The Alzheimer's Association (Rafii et
al., 2013), were presented by Raymond Bartus, Ph.D., who led the preclinical
and early clinical development of CERE-110.
It is well-documented that cholinergic nerve cells in the brain degenerate
early in AD and are linked to initial memory loss and cognitive decline.
Current therapies for AD include a class of small molecule drugs called
cholinesterase inhibitors, which aim to preserve or enhance the chemical
signaling in cholinergic nerves. However, these drugs have dose-limiting
side-effects, do not change the underlying disease process or its progression,
are effective for some but not all people, and may help only for a limited
time.
Research in animal models suggests that growth factors, such as NGF, which
promote nerve growth, nerve repair and protect nerves against damage, may
improve AD symptoms and slow disease progression. However, delivery of
proteins to the appropriate part of the brain, in a manner that enables them
to have a therapeutic effect, has proved challenging due to the blood brain
barrier. Gene therapy with AAV, combined with stereotactic surgery, enables
delivery of the gene encoding a biologically active protein such as NGF to a
precise location in the brain. The nucleus basalis of Meynert (NBM), a
structure near the base of the brain, is considered to be the best target for
delivery of a gene-based therapeutic for AD as its cells represent the primary
source of cholinergic nerves projecting to the brain cortex. Thus, a single
treatment of CERE-110 into this structure is expected to provide a safe
long-term source of NGF protein that may protect and preserve cholinergic
cells in the brains of AD patients and potentially slow disease progression.
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