CytoTherapeutics` Study in
Nature Demonstrates
Neuroprotection in Primate
Model of Huntington`s Disease
Encapsulated-Cell Delivery of
CNTF Shown to Prevent Damage
to Brain Circuitry <>
PROVIDENCE, R.I., March 26 /PRNewswire/ via Individual Inc. --
CytoTherapeutics, Inc. (Nasdaq: CTII) reported today that
administration of a neurotrophic factor via the Company's
encapsulated-cell system was shown to be neuroprotective in a
non-human primate model of Huntington's disease. Researchers
demonstrated that human CNTF (hCNTF), delivered in this
manner, significantly reduced the striatal neuron loss which is
associated with movement abnormalities, as well as reducing the
damage of a key region within the cerebral cortex. As reported by
Dwaine F. Emerich, Ph.D. and colleagues at CytoTherapeutics,
and Jeffrey H. Kordower, Ph.D. of Rush Presbyterian Medical
Center in the current issue of Nature, these results represent the
first primate study to show definitively that a growth factor can
act as a potential therapeutic agent in protecting multiple neuronal
populations in an animal model of Huntington's disease. The
research published in today's Nature was sponsored by
CytoTherapeutics of Providence, Rhode Island.
"We are optimistic about the results of our Huntington's disease
study published in Nature. Delivery of growth factors based on
this cell- encapsulated technology has provided us with a new
avenue for neuroprotective therapy," stated Dr. Kordower. "This
technology is particularly exciting for patients with Huntington's
disease. Through genetic testing, all those at risk for Huntington's
disease can be identified decades prior to the onset of symptoms
or neural degeneration. Thus, unlike Parkinson's or Alzheimer's
disease, where extensive degeneration has already taken place at
the time of clinical diagnosis, we can potentially intervene using
this technology well before neural degeneration takes place --
optimizing our chances to provide neuroprotection for these
patients."
In the study, human CNTF was found to preserve several
populations of striatal cells, including GABAergic, cholinergic
and diaphorase-positive neurons, in addition to critical
non-striatal regions associated with cognitive capabilities.
Researchers utilized CytoTherapeutics' cell- containing devices to
deliver hCNTF. The model used was based on the injection of
quinolinic acid (QA) into the striatal region of the primates' brains
to produce death of the specific neurons similar to those lost in
Huntington's disease. Prior to the unilateral QA lesioning,
CytoTherapeutics' devices were implanted into brain tissue of
non-human primates. Animals receiving the hCNTF-secreting
devices exhibited a significant sparing of the neuronal cells that
would normally have been lost following the lesion.
"The results of the study reported today in Nature are very
encouraging. These data support the therapeutic rationale for use
of our encapsulated-cell devices to deliver, site specifically, a
continuous dose of a potentially neuroprotective agent in
Huntington's disease," said Sandra Nusinoff Lehrman, M.D.,
President and Chief Operating Officer. "To date, there is no
effective treatment capable of slowing or preventing the course of
this devastating disease. We hope that our approach will offer a
vital new avenue to researchers in this field."
Growth factors, alone and in combination, are believed to have
substantial potential for halting the progressive loss of neuronal
tissue characteristic of many central nervous system, or CNS,
disorders. CytoTherapeutics is investigating the potential use of
its encapsulated-cell products to deliver additional growth factors
to treat Huntington's disease. The Company believes that its
technology may represent a practical means of long-term delivery
of such growth factors across the blood-brain barrier, avoiding
many of the side- effects associated with current methods for
delivering these substances within the CNS.
The current study of the delivery of growth factors to treat
Huntington's disease builds on work by Drs. Emerich and
Kordower. As previously reported in the Journal of Neuroscience
in 1996, delivery of hCNTF reduced both loss of specific neurons
and subsequent movement abnormalities in a rodent model of
Huntington's disease. In 1994, as reported in Experimental
Neurology, researchers employed the same rodent model for
Huntington's disease to deliver human nerve growth factor
(hNGF). In this study, hNGF administered directly to the CNS of
rodents produced sparing of a specific neuronal population and a
corresponding reduction in movement abnormalities.
Huntington's disease, which affects more than 25,000 patients in
the United States, is a genetically determined, progressive
neurodegenerative disease for which there is no cure. The
movement disorders and cognitive losses that are symptomatic of
the disease are caused by the death of neurons within the striatal
and critical non-striatal regions of the brain.
CytoTherapeutics, Inc. is a leader in the development of novel cell
therapy systems designed to deliver therapeutic substances to
the central nervous system (CNS). The Company's CNS-focused
technology is designed to provide controlled, site-specific and
safe delivery of a variety of novel therapeutic substances across
the blood-brain barrier, potentially overcoming a fundamental
obstacle in the effective treatment of CNS diseases. At its
headquarters in Providence, Rhode Island, the Company is
currently developing products for the treatment of chronic pain,
ALS, Parkinson's disease and Huntington's disease, with
additional efforts directed to several other CNS disorders.
Through its 50%-owned Swiss subsidiary, Modex Therapeutiques
SA, the Company is also developing applications of its
technology to treat diabetes, blood disorders and obesity. For
additional information, visit the Company's Internet web site at
http://www.cyto.com.
Certain statements in this press release include forward-looking
comments regarding, among other things, product development
programs and potential new products. CytoTherapeutics' actual
results may vary materially from those forward looking statements
due to risks and uncertainties, such as the need to inlicense rights
to hCNTF or other neurotrophic factors, delays in obtaining
regulatory approvals, uncertainties of product development, or
delays in product development and other risks to which the
Company is subject and which are described in Exhibit 99 to the
Company's Annual Report on Form 10-K entitled "Cautionary
Factors Relevant to Forward Looking Statements."
SOURCE CytoTherapeutics, Inc.
/CONTACT: Seth A. Rudnick, M.D., Chairman and CEO or Sandra
Nusinoff Lehrman, M.D., President and COO of
CytoTherapeutics, Inc., 401-272-3310, or Karen L. Bergman
(media) or Reagan Codner (investors) of Burns McClellan, Inc.,
212-505-1919/ (CTII)
[Copyright 1997, PR Newswire]
