Treating Familial Hypertrophic Cardiomyopathy (HCM) by Throttling Back the Heart’s Molecular Motor; Possible Myosin Inhibitor Treatment Builds on Key Advance Made 15 Years Ago; Condition Often Culprit in Sudden Death of Young Athletes

More than 15 years ago, David Warshaw, Ph.D., and coworkers discovered the precise malfunction of a specific protein in the heart that leads to hypertrophic cardiomyopathy (HCM), a common culprit in cases of sudden death in young athletes. Now, a team of scientists has used some of Dr. Warshaw's earlier findings to develop a possible treatment to prevent HCM, an inherited disease that can cause the heart to thicken and stop pumping blood effectively, leading to heart failure. Dr. Warshaw, now Professor and Chair of Molecular Physiology and Biophysics at the University of Vermont (UVM) College of Medicine, wrote about the significance of this potential therapy in a "Perspectives" column in the February 5, 2016 issue of the journal Science. The title of this piece is “Throttling Bace the Heart’s Molecular Motor.” "This may offer a generalized approach to solving hypertrophic cardiomyopathy," says Dr. Warshaw, who is also an Investigator in the Cardiovascular Research Institute of Vermont at UVM. "I think it's extremely promising." HCM can result from different mutations of many different proteins in the heart. One of those proteins, myosin, acts as a tiny molecular motor in every heart muscle cell. Myosin pulls on and releases a rope-like protein, actin, in order to make the heart muscle contract and relax as it pumps blood. A mutation of myosin can "alter the motor's power-generating capacity" and make the heart work improperly, which in turn causes the heart to enlarge (hypertrophy), Dr. Warshaw says. For many years, scientists assumed that the mutation caused the myosin to lose its motoring power, throwing off the whole heart engine. But in a study Dr.
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