Experimental drug reverses effects of Fabry disease in mice

Shows promise for related disorders

ANN ARBOR, 12 June 2000 -- A new experimental drug developed at the University of Michigan Medical School is the first treatment shown to reverse the effects in mice of a hereditary, incurable disorder called Fabry (Fah-BRAY) disease, which affects between 6,000 and 10,000 men in the United States.

Results of the study were published in the June 2000 issue of the Journal of Clinical Investigation in an article by James Shayman, M.D., U-M professor of pharmacology and of internal medicine, and colleagues from the U-M and the National Institutes of Health.

Fabry disease is caused by a genetic mutation that prevents production of an enzyme called alpha-galactosidase A, which cells store in tiny globules called lysosomes. The cellular version of a garbage disposal, lysosomes are found in all cells that use enzymes to dissolve specific molecular bonds and digest waste products.

Without alpha-galactosidase A, lysosomes cannot break up one particular type of glycolipid, a long chain of fats and sugars found in cell membranes. As a result, these molecules accumulate within the kidneys, hearts and blood vessels of patients with Fabry disease. Death occurs in early adulthood from renal failure or cardiovascular complications.

For the past 10 years, U-M researchers have been developing and perfecting a family of glycolipid synthesis inhibitors for use in Fabry disease, according to Shayman. "Other researchers tried to replace the missing enzyme, but we concentrated on blocking glycolipid formation by inhibiting a key enzyme the cell needs to produce these molecules. We now have two compounds with high levels of inhibitory activity and no observed toxic side effects."

According to the Journal of Clinical Investigation study, treatment with one of these compounds produced a highly significant reduction in glycolipid levels in kidneys of male mice with Fabry disease. When treated for eight

Contact: Sally Pobojewski
University of Michigan

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