REGENERON PHARMACEUTICALS DEMONSTRATES IN ANIMAL STUDIES THAT
NERVE GROWTH FACTOR CNTF SLOWS PROGRESSION OF LOU GEHRIG'S DISEASE
NEW ORLEANS, Nov. 12 /PRNewswire/ -- For the first time, scientists have demonstrated in animal studies that a biotechnology-based nerve growth factor can promote the survival of motor neuron cells that degenerate and die in people with amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig's disease. Scientists from Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN), announced the successful results of their animal studies of ciliary neurotrophic factor (CNTF) at the Society for Neuroscience meeting here today.
"We believe this is an important advance toward developing a treatment for ALS, a devastating disease for which there is no known cure," said Ronald M. Lindsay, program director of the neurobiology group for Regeneron Pharmaceuticals.
"Our study was conducted on Mnd mice, a strain with a naturally occurring motor neuron deficit very similar to the one in people with ALS," Lindsay said. "As with ALS victims, the Mnd mice exhibit rapid deterioration of muscle control, limb coordination and the ability to walk once the disease takes hold, and eventually die prematurely.
"When we treated the Mnd mice with injections of CNTF over a two-month period, we found that progression of the disease slowed significantly," Lindsay continued. "There were no significant side- effects. We are continuing animal testing of this biotechnology-based compound, and are laying the groundwork for multi-center clinical trials in humans. By the middle of next year, we hope to seek FDA approval to begin clinical testing for the potential treatment of ALS."
Regeneron's treatment of the Mnd mice began at five months, the time when symptoms of the disease typically begin to appear, Lindsay said.
Using footprint analysis to measure progression of the disease, Maureen Helgren at Regeneron, a specialist in animal behavior, has shown that the stride length and inter-step distance of Mnd mice decreases by 30-40 percent during the first eight to 12 weeks of the disease. Treatment of Mnd mice with CNTF during this period markedly slows the progression of these characteristic deficits in walking behavior. These results were graphically shown at the Society for Neuroscience meeting in a videotape of treated and untreated Mnd mice.
Lindsay cautioned that there is a need for additional testing and compliance with FDA statutes and regulations before CNTF can be evaluated in humans or marketed as a commercial product. He said that Regeneron could not estimate whether, or when, any commercial products resulting from these trials could be on the market.
Early Development of CNTF
In May 1990, in the scientific journal Nature, Regeneron's scientific collaborators at the Max Planck Institute in Munich, Germany, were the first to report direct beneficial effects of CNTF on motor neurons following their injury in animals. Building on these findings, Regeneron scientists and scientific collaborators have continued to research the properties of CNTF and to accumulate data in animal models.
Regeneron scientists described in a July 1991 article in Science how they successfully cloned the receptor for CNTF -- a vital link in the nervous system's ability to use the neurotrophic factor. The identification and cloning of receptors are significant steps in the development of neurotrophic factors as product candidates, and can be used to identify new and unpredicted uses of the factors.
Regeneron scientists also reported in the July 1991 Science article that the CNTF receptor is present in muscle tissue, indicating the possibility that it might be useful in the treatment of muscle diseases. The company is conducting preclinical evaluations of CNTF to further understand their potential use.
Leader in Developing Treatments for
Regeneron Pharmaceuticals, Inc., based in Tarrytown, N.Y., is a leader in the discovery and development of biotechnology-based compounds for the treatment of neurodegenerative diseases, peripheral neuropathies and nerve injuries, which afflict more than seven million Americans. In addition to CNTF, the company is jointly developing two neurotrophic factors, brain-derived neurotrophic factor (BDNF) and neurotrophin-3 (NT-3), with Amgen, Inc. Regeneron also has an exclusive license to a fourth factor, neurotrophin-4 (NT-4), which was recently discovered by scientists at the Karolinska Institute in Stockholm, Sweden.
Regeneron discussed continued progress in the preclinical development of each of its neurotrophic factors at the Society for Neuroscience meeting.
/CONTACT: Fredric Price of Regeneron, 914-347-7000, or Michael Gross, 212-484-7721, or Karen Barzilay, 212-484-7465, both of Robinson, Lake, Lerer & Montgomery, for Regeneron/
(REGN) CO: Regeneron Pharmaceuticals, Inc. ST: New York, Louisiana IN: MTC SU: GK-OS -- NY027 -- 3405 11/12/91 11:09 EST