Lahey Hospital Research Trial Supports Patients With Heart Conditions

BURLINGTON, MA — A new therapy for patients experiencing a form of genetic heart disease underwent a trial led by a director at Lahey Hospital & Medical Center in Burlington.

One of the most common genetic heart diseases worldwide, hypertrophic cardiomyopathy causes the walls of the left ventricle to become thick and stiff. In about 70 percent of cases, patients with this condition experience obstruction to blood flow, which increases pressures in the heart and can lead to chest pain, shortness of breath, and reduced exercise capacity.

A trial of a drug called aficamten leads "to significant relief in the burden of limiting symptoms, improving the quality of patients' lives," according to a statement from the hospital.

Martin S. Maron, director of the Hypertrophic Cardiomyopathy Center at the hospital, was principal investigator of the research, which was published in the New England Journal of Medicine.

“The trial demonstrated that aficamten can reliably and safely lower obstruction to blood flow out of the heart in patients (experiencing this condition) using a simple and stepwise dosing regimen and was associated with substantial improvements in clinically relevant endpoints such as exercise capacity and symptoms,” said Maron. “These patients are often on a multitude of medications, and aficamten was effective as combination therapy, but also as monotherapy.”

The trial included 282 patients with hypertrophic cardiomyopathy from 101 sites in 14 countries in North America, Asia, and Europe, making it the largest trial ever such trial for patients with this condition.

Over the 24-week treatment period, aficamten improved exercise capacity and was also associated with significant improvements in other outcomes.

The full research is available at the New England Journal of Medicine.