PerspectiveHeart Disease

Throttling back the heart's molecular motor

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Science  05 Feb 2016:
Vol. 351, Issue 6273, pp. 556-557
DOI: 10.1126/science.aaf1636

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A young athlete collapses and dies during competition. Autopsy reveals an enlarged heart with thickened walls in which the cardiac muscle cells are in disarray and surrounded by fibrotic tissue. Until 1990, the cause of such sudden death was unknown. This devastating condition, called familial hypertrophic cardiomyopathy (HCM), was eventually linked to a mutation in myosin (1), the heart's molecular motor. Today, more than 300 separate HCM-causing mutations have been identified throughout the myosin molecule. On page 617 of this issue, Green et al. (2) describe a small molecule that binds to myosin and inhibits its activity, delaying the onset and progression of the disease in a mouse model. The study offers hope that a “simple” remedy for HCM may be possible.