Researchers identify possible drug target for treating dilated cardiomyopathy

Researchers are reporting identification of a possible drug target for treating dilated cardiomyopathy, a potentially deadly condition involving enlargement of the heart’s main pumping chamber.

In dilated cardiomyopathy, the heart’s main pumping chamber expands so much that the heart can no longer beat regularly. People with the condition experience shortness of breath, chest pain, and in severe cases, sudden and deadly cardiac arrest. About 1 in every 250 Americans suffer from a form of dilated cardiomyopathy. Its exact cause is unknown, but it appears to have a genetic component.

The researchers studied heart muscle cells grown from people with a genetic mutation associated with dilated cardiomyopathy. They found that the mutation appears to activate a biological pathway normally turned off in healthy adult hearts, causing the heart cells to beat improperly. In lab studies, the researchers showed that chemically inhibiting the pathway corrected the mutation’s effects, restoring regular beating of the heart cells. The researchers accomplished this using drugs that are already approved by the Food and Drug Administration.

The study, partly funded by NHLBI, appeared in Nature.

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