I thought this would be of interest to the PD community.
Somatix scientist presents Parkinson's data at Fifth
InternationalNeurotransplantation Symposium ALAMEDA, CALIF. (JUNE 28)
BUSINESS WIRE - June 28, 1994--Somatix Therapy Corporation (NASDAQ: SOMA)
announced today that Dr. Krzysztof Bankiewicz, Ph.D., section head of
preclinical studies in the company's neural diseases program, presented
preclinical data at the Fifth International Neurotransplantation Symposium in
Paris.
In his presentation, entitled "Clinical Recovery and Graft Survival in
Parkinsonian Non-Human Primates After Striatal Grafting of Fibroblasts
Genetically Modified with Tyrosine Hydroxylase cDNA," Bankiewicz described a
pilot experiment showing reversal of parkinsonian symptoms in non-human
primates after the implantation of genetically modified cells.
The experiment, which was primarily designed to test transplantation
methodologies and safety, incorporated various measures of efficacy including
behavioral changes and biochemical analysis.
Seven animals were used, all of which had been chemically treated to exhibit
severe Parkinson's symptoms. One group of 3 animals received genetically
altered cells, another group of 2 animals received unmodified cells and a
third group received no implants.
The group of animals that received the genetically modified cells showed
improvement in motor skills within 24 hours of implantation. A number of
behavioral symptoms indicative of Parkinson's disease, including tremors,
posture and gait, which were present in the animals before treatment were no
longer exhibited. The animals in the control groups showed no similar
changes.
The implantation of genetically modified cells in the brain is a therapeutic
strategy being developed by Somatix for the treatment of Parkinson's disease,
a disease caused by the loss of dopamine-producing neurons. The goal of
Somatix's therapy is the sustained delivery of therapeutic levels of dopamine
within the blood-brain barrier.
In the proposed approach, autologous skin cells would be removed from the
patient and genetically modified to produce tyrosine hydroxylase (TH). TH
converts the common amino acid tyrosine to L-dopa which is subsequently
converted to dopamine. Oral, systemic administration of L-dopa is the
mainstay of current Parkinson's therapy, however, it is toxic at high doses
and can generate serious side effects.
Somatix's scientists believe that production of L-dopa inside the blood-brain
barrier will be far less toxic than the current method of oral L-dopa
administration and may eliminate the side effects.
Bankiewicz presented additional data indicating that the genetically altered
cells were still healthy after four months, the time at which the animals
were sacrificed, and were still producing TH.
Dr. Bankiewicz joined Somatix in August 1992 after working in the neurology
branch of the National Institutes of Health (NIH) since 1985. While at the
NIH, he designed brain implantation studies and established a primate model
that mimics Parkinson's disease in humans. This primate model was used in
the experiments.
Somatix Therapy Corporation is a leader in the field of gene therapy. The
company intends to research, develop and commercialize proprietary processes
for the genetic modification of cells and their use in the treatment of human
disease. Somatix's assets include its highly efficient gene transfer
technology, broad-based intellectual property and product development
programs focused on cancer, neural diseases and hemophilia.
--30--pc/sf*
CONTACT: Somatix Therapy Corp.
Mark Bagnall, 510/748-3064
KEYWORD: CALIFORNIA
INDUSTRY KEYWORD: MEDICINE PHARMACEUTICAL BIOTECHNOLOGY REPEATS: New York
212-575-8822 or 800-221-2462; Boston 617-330-5311 or 800-225-2030; SF
415-986-4422 or 800-227-0845; LA 310-820-9473
Transmitted: 94-06-28 09:19:37 EDT
