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Compound Designed by Vertex Pharmaceuticals Accelerates Nerve Regeneration in Rat Model Report at the Society for Neuroscience 26th Annual Meeting Source: PR Newswire CAMBRIDGE, Mass., Nov. 18 /PRNewswire/ via Individual Inc. -- Researchers at Vertex Pharmaceuticals (Nasdaq: VRTX) have designed an orally administerable compound that in a rat model accelerates functional recovery and nerve regeneration following nerve injury. The results obtained in the rat model were presented yesterday by Dr. Bruce Gold, Associate Professor, Department of Cell and Developmental Biology of the Oregon Health Sciences University, at the Society for Neuroscience 26th Annual Meeting being held this week in Washington, DC. "Neurodegenerative diseases and nerve injury are among the most debilitating medical conditions. In clinical trials, genetically engineered growth factor proteins and peptides have shown promise as treatments to regenerate nerves. However, protein-based drugs have significant limitations, including intravenous dosing, poor pharmacokinetics, and limited penetration and activity in the central nervous system," explained Dr. Vicki Sato, Senior Vice President of Research and Development and Chief Scientific Officer of Vertex. "Vertex's research has been directed toward designing orally administered compounds that would enhance the functional recovery of injured neurons. The research reported today shows that a representative compound from a bank of compounds designed by Vertex accelerates nerve repair in a rat model of nerve injury." In the rat model, controlled injury to the sciatic nerve, which extends from the hip through the leg, rendered the rat unable to walk. The researchers evaluated the ability of Vertex's compound to accelerate nerve regeneration in this model. Vertex's compound accelerated the onset of foot movement and walking compared to the control. In addition, Vertex's compound produced a 50 percent increase in the average size of the axons in the injured area as compared to the control animals. In his presentation, Dr. Gold reported that Vertex's compound mimics the nerve repair activity of the immunosuppressive drug FK506, without suppressing the immune system. He also reported that the mechanism of nerve repair displayed by Vertex's compounds and FK506 is independent of inhibition of calcineurin, a signaling molecule and a biological target for FK506. The results observed with Vertex's compound in the rat model were comparable to the nerve repair activity Dr. Gold has observed for FK506. In 1993, Dr. Gold was first to report the nerve regeneration activity of FK506. Dr. David Armistead, Senior Scientist at Vertex, commented, "As the next step in our research, the activity of our compounds is being assessed in models of neurodegenerative disorders, such as Alzheimer's and Parkinson's diseases, and in additional models of peripheral neuropathies. We are also conducting experiments that may provide insight into defining the molecular mechanism by which our compounds exert their nerve growth effect." Dr. Armistead cautioned, "The findings reported today are preliminary and it will be years before we will know whether or not these compounds have therapeutic benefit in patients with nerve injury." The goal of Vertex's neuroimmunophilin program is to design and develop orally administered compounds to treat neurodegenerative diseases, such as Alzheimer's and Parkinson's diseases; peripheral neuropathies, which result from degeneration of sensory and motor nerves; as well as to treat nerve injury resulting from trauma or stroke. Vertex has been granted three patents containing claims related to the Company's neuroimmunophilins compounds by the United States Patent and Trademark Office. Additional patent applications are pending in the United States and in other countries worldwide. Vertex Pharmaceuticals Incorporated is engaged in the discovery, development and commercialization of novel, small molecule pharmaceuticals for the treatment of diseases for which there are currently limited or no effective treatments. The Company is a leader in the use of structure-based drug design, an approach to drug discovery that integrates advanced biology, biophysics and chemistry. The Company is concentrating on the discovery and development of drugs for the treatment of viral diseases, multidrug resistance in cancer, hemoglobin disorders, inflammation, autoimmune diseases, organ transplant rejection and neurodegenerative diseases. SOURCE Vertex Pharmaceuticals Incorporated /CONTACT: Lynne H. Brum, Director of Corporate Communications of Vertex 617-577-6000 or http://www.vpharm.com/ [11-18-96 at 08:29 EST, PR Newswire]