Under a new $4.7 million grant from the National Eye Institute, researchers from Case Western Reserve University School of Medicine and University Hospitals of Cleveland hope to develop in five years a drug ready for human clinical trials to treat myasthenia gravis, an autoimmune disease that weakens muscles, affects vision, and in the most severe cases, puts patients on artificial ventilation. According to the National Eye Institute, this is the largest single project grant it has awarded to an Ohio institution.

The new grant to Henry J. Kaminski, M.D., a professor in the Department of Neurology and a member of the Visual Sciences Research Center at Case and UHC, brings together a group of experts well-versed in the disease, muscle biology, genetics, and drug development who are associated with Case, UHC and the Louis Stokes Cleveland Department of Veterans Affairs Medical Center.

Among the unique features of this research project is that it will be the first to apply genomic analysis to this disorder and will be the first to comprehensively investigate eye muscle involvement by the disease.

In autoimmune diseases, the immune system malfunctions and mistakes a part of the body as foreign and mounts an attack on it. According to Kaminski, in myasthenia gravis, the immune system produces antibodies that attack the receptors on the muscle side of the neuromuscular junction. The antibodies cause damage by activating another part of the immune system called the complement system, which is a group of proteins designed to protect against infectious invaders.

"We hope to design a drug that inhibits complement activation specifically at the nerve-muscle communication point," said Kaminski. "Although there currently are several treatments that are beneficial in treating the disease, they have numerous adverse effects. We hope our treatment would be a more effective one with less side effects."

Kaminski said that although the
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Contact: George Stamatis
George.Stamatis@case.edu
216-368-3635
Case Western Reserve University
10-Nov-2003

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