Researchers find inhibitor that helps prevent hypertrophic cardiomyopathy in mice

16th February 2016

Researchers say they may have pinpointed a treatment that might prevent hypertrophic cardiomyopathy (HCM).

Using research from 15 years ago that showed the condition can be caused by problems in the myosin protein, the team has been testing a small molecule inhibitor on mice with myosin gene mutations.

Myosin is responsible for making the heart muscle contract and relax as it pumps blood. A mutation of myosin can damage the heart’s “power-generating capacity” and cause it to enlarge.

In their study, reported in Science, the researchers found the inhibitor altered the myosin motor’s power generation to a more normal level. They gave the drug containing the molecule to the mice as early as eight weeks old, and found that it prevented the HCM from surfacing, said the team.

When they gave the drug to a young mouse with the mutation, the mouse’s heart developed normally.

Because HCM runs in families, a young child who tests positive for the genetic mutation could receive the treatment to help stave off the disease. However, development of a human drug would require much more extensive testing.

The researchers are from Harvard Medical School, Stanford University School of Medicine, University of Colorado, and drugs company MyoKardia.

For more details, see here.