Sep. 12, 1998 CINCINNATI - The drug cyclosporin may be effective in treating hypertrophic cardiomyopathy (HCM), a heart disease cited as the most frequent cause of sudden death in young adults and young athletes, according to a new study by researchers at Children's Hospital Medical Center of Cincinnati.
The study, to be published in the September 11 edition of Science, shows that cyclosporin, a drug commonly used to prevent rejection of transplanted organs, prevents HCM in rats and mice. The study was led by Jeffery D. Molkentin, Ph.D., a researcher in the division of molecular cardiovascular biology and a Pew Scholar at Cincinnati Children's.
HCM is an inherited form of heart disease that affects one in 500 individuals. The disease starts as a general enlargement of the heart's middle muscular layer and can ultimately progress to heart failure. Several forms of inherited heart disease, including HCM, are associated with genetic mutations that cause thickening of the heart muscle wall. This thickening is known as cardiac hypertrophy.
In a previous study, published in April in the journal Cell, Dr. Molkentin and his colleagues showed that the enzyme calcineurin can initiate cardiac hypertrophy in mice. In the new study, the scientists administered a calcineurin inhibitor, cyclosporin, to mice that were genetically predisposed to develop HCM and to rats in which HCM was induced. This prevented the development of HCM.
"These results suggest that calcineurin inhibitors merit further study as potential treatments for certain forms of human heart disease," says Dr. Molkentin. "Because long-term use of cyclosporin may have adverse side effects, different calcineurin inhibitors may need to be developed."
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