Alexion-Yale Repair Spinal Cord Damage in Animal Model by Xenotransplantation of Transgenic Pig Neurons Studies Reported at 28th Meeting of Society for Neuroscience


Nov 12 1998


LOS ANGELES, Nov. 12 - PRNewswire - Researchers from the Yale University School of Medicine and Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) are presenting a poster at the 28th Meeting of the Society for Neuroscience here today that reports on novel approaches to the transplantation of pig cells which have implications for the treatment of spinal cord injury patients. The report includes data showing that the transgenic pig cells form a sheath around damaged neurons in animals whose spinal cords were surgically severed, and that the spinal cords that had received the pig cell transplants showed restoration of normal nerve signal conduction.

"We are very excited by these results demonstrating that cells from Alexion's patented transgenic complement resistant pigs facilitate both nerve regeneration and the restoration of spinal cord conduction in this animal model of spinal cord injury," said Leonard Bell, M.D., President and Chief Executive Officer of Alexion. "We look forward to the day when we will be able to provide a ready source of cells with the potential to restore function to patients suffering from paralysis due to spinal cord injury."

Entitled "Facilitation of Axonal Conductance Across Transected Rat Spinal Cord Following Transplantation of Olfactory Ensheathing Cells or Schwann Cells" the report comes from the laboratories of Dr. Jeffery D. Koesis of the Department of Neurology, Yale University School of Medicine, and Dr. William L. Fodor, Senior Director of Xenotransplantation, Alexion Pharmaceuticals, Inc. and their colleagues. Both Yale and Alexion are located in New Haven, CT.

"Alexion's transgenic pig cells were associated with the highest levels of spinal cord repair and regeneration that we have yet seen using this transplantation approach," said Dr. Koesis. "We have also started to test these cells in primates, and are encouraged by preliminary results obtained with transgenic pig cells that look similar to the results obtained in rats."

According to the Christopher Reeve Foundation, more than half of the spinal cord injured individuals in the United States were injured between the ages of 16 and 30, with the majority (90%) of people surviving and living near normal lifespans.

"We are encouraged by Alexion's results particularly as they relate to olfactory ensheathing cells (0EC)," said Mr. Augusto Odone, President of the Myelin Project in Washington, D.C. "Studies performed by Myelin Project researchers on both sides of the Atlantic have shown that these cells not only promote axonal regrowth, but also foster remyelination of naked axons. The availability of porcine OEC transplantable into the human central nervous system (CNS) may obviate the difficulties of obtaining significant amounts of these cells from humans. Alexion's xenotransplantation technology may thus greatly contribute to ongoing efforts to remyelinate the CNS of Multiple Sclerosis and leukodystrophy sufferers."

Augusto and Michaela Odone's only child, Lorenzo, is afflicted with a hereditary demyelinating disease, adrenoleukodystrophy (ALD). Although not medical doctors, the Odones developed a therapy, now adopted on both sides of the Atlantic, which reverses the biochemical defect of ALD. The story of the Odone's struggle against ALD was dramatized in the 1992 Universal Studios release Lorenzo's Oil starring Nick Nolte and Susan Sarandon.

According to the National Spinal Cord Injury Association, there are approximately 8,000 new spinal cord injury patients in the U.S. each year and at least 200,000 patients have suffered a traumatic spinal cord injury. Most spinal cord injury patients are young adults and slightly over one-half of patients suffer the most severe form of non-fatal spinal injury, quadriplegia, or loss of use of all four limbs. "Alexion's findings represent an important stepping stone toward the overall management of spinal cord injury patients," said Thomas H. Countee, Jr., Executive Director and Chief Executive Officer of the National Spinal Cord Injury Association.

Xenotransplantation refers to the transplantation of non-human cells, tissues and/or organs into human patients. The genetic engineering of pigs with cells expressing human complement inhibitors may provide a highly effective means of protecting the cells, tissues and organs of such pigs from rejection, thereby providing more readily accepted xenotransplantation products.

Alexion is pioneering a double-barreled approach to the problem of complement- mediated rejection of xenografts. This proprietary approach involves the creation of transgenic pigs that have been genetically altered to express human complement inhibitor proteins (which can provide a shield against complement-mediated rejection), and to reduce or eliminate the expression of certain sugar structures that can trigger complement-mediated rejection.

Alexion Pharmaceuticals, Inc. was founded in 1992 and is engaged in the development of selective immunotherapeutic drugs that generally are designed to inhibit the disease-causing segments of the immune system while preserving the disease-preventing aspects of the immune system. The Company is developing three technology platforms: C5 Complement Inhibitors and Apogen T- Cell Therapeutics which together target severe cardiovascular and autoimmune disorders; and xenografts for organ transplantation.

This news release contains forward-looking statements. Such statements are subject to certain factors which may cause Alexion's plans to differ or results to vary from those expected including unexpected pre-clinical or clinical results, the need for additional research and testing, delays in manufacturing, access to capital and funding, delays and adverse changes in development of commercial relationships and a variety of risks set forth from time to time in Alexion's filings with the Securities and Exchange Commission, including but not limited to the risks discussed in Alexion's Annual Report on form 10-K for the year ended July 31, 1998. Alexion undertakes no obligation to publicly release results of any of these forward-looking statements which may be made to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. SOURCE Alexion Pharmaceuticals, Inc.


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