A small-molecule inhibitor of sarcomere contractility suppresses hypertrophic cardiomyopathy in mice

EM Green, H Wakimoto, RL Anderson, MJ Evanchik… - Science, 2016 - science.org
EM Green, H Wakimoto, RL Anderson, MJ Evanchik, JM Gorham, BC Harrison, M Henze…
Science, 2016science.org
Hypertrophic cardiomyopathy (HCM) is an inherited disease of heart muscle that can be
caused by mutations in sarcomere proteins. Clinical diagnosis depends on an abnormal
thickening of the heart, but the earliest signs of disease are hyperdynamic contraction and
impaired relaxation. Whereas some in vitro studies of power generation by mutant and wild-
type sarcomere proteins are consistent with mutant sarcomeres exhibiting enhanced
contractile power, others are not. We identified a small molecule, MYK-461, that reduces …
Hypertrophic cardiomyopathy (HCM) is an inherited disease of heart muscle that can be caused by mutations in sarcomere proteins. Clinical diagnosis depends on an abnormal thickening of the heart, but the earliest signs of disease are hyperdynamic contraction and impaired relaxation. Whereas some in vitro studies of power generation by mutant and wild-type sarcomere proteins are consistent with mutant sarcomeres exhibiting enhanced contractile power, others are not. We identified a small molecule, MYK-461, that reduces contractility by decreasing the adenosine triphosphatase activity of the cardiac myosin heavy chain. Here we demonstrate that early, chronic administration of MYK-461 suppresses the development of ventricular hypertrophy, cardiomyocyte disarray, and myocardial fibrosis and attenuates hypertrophic and profibrotic gene expression in mice harboring heterozygous human mutations in the myosin heavy chain. These data indicate that hyperdynamic contraction is essential for HCM pathobiology and that inhibitors of sarcomere contraction may be a valuable therapeutic approach for HCM.
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