Experimental drug reverses effects of Fabry disea...( weeks with twice-daily injections glyc...),Health

weeks with twice-daily injections, glycolipid levels after treatment were lower than they were before treatment began---a result Shayman described as the most unexpected in the study. Equally important, the mice did not lose weight or develop toxic side effects from therapeutic levels of the inhibiting compound.

"Now that we know it works in mice, our next step is pre-clinical drug development," Shayman says. "We hope to show efficacy with oral intake, so the drug will not need to be given intravenously. Then, we plan to apply for Investigational New Drug approval and orphan disease designation from the Food and Drug Administration prior to the initiation of clinical trials."

Fabry disease is called an "orphan disease," because it is so rare. According to Shayman, however, six other lysosomal storage diseases are closely related to Fabry and should be treatable with the same approach. Together these seven diseases affect between 20,000 and 30,000 people in the United States. Although caused by different missing enzymes, all seven diseases are characterized by accumulations of similar types of glycolipids in different organs in the body. Since the U-M compound inhibits production of these glycolipids, Shayman plans to test its effectiveness in these related disorders, which include Gaucher disease, Tay-Sachs disease, Sandhoff disease and GM1 gangliosidosis.

"Currently only Gaucher disease has an approved therapy, which involves regular intravenous infusions of a genetically engineered form of the missing enzyme called recombinant beta-glucosidase," Shayman says. "It costs more than $200,000 for one year's treatment and currently is the most expensive drug on the market."

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Contact: Sally Pobojewski
pobo@umich.edu
734-647-1844
University of Michigan
11-Jun-2000

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