New combination of drugs slows heart decline in muscular dystrophy patients

Dr Subha Raman led a team of scientists in exploring the effects of combining heart failure drugs to treat sufferers of Duchenne muscular dystrophy

Signs of Duchenne muscular dystrophy (DMD) can start to appear in boys as young as six, leading to deterioration of the heart muscles and ultimately death. Pharmaceuticals aimed at controlling high blood pressure have been used to treat the one in 3,500 young males suffering from the condition, but a new study suggests that a novel combination of these drugs could slow the decline in heart function earlier on, and in promising new ways.

Led by cardiologist Dr Subha Raman, a professor at Ohio State University Wexner Medical Center, a research team explored the effects of combining heart failure drugs to treat heart muscle disease over a twelve month period. A group of 42 boys suffering from DMD and heart muscle degeneration were treated with either an ACE inhibitor or an angiotensin receptor blocker (ARB), drugs used to treat elevated blood pressure.

Among the 42 boys, a random group was also treated with eplerenone, another blood pressure medication, while the rest were given a placebo. The subjects underwent cardiac MRIs before, halfway through and then following the 12 month study period, enabling the researchers to track the condition of their cardiovascular health.

The researchers reported that the patients receiving eplerenone displayed less of a decline in the function of the left ventricle, the chamber of the heart responsible for pumping oxygenated blood to tissue around the body. Changes to this function of the heart occur well before more severe symptoms of DMD, such as heart failure or fatal arrhythmias, giving the researchers hope of targeting heart degeneration early on.

The findings indicated to Raman that at least six months of the treatment would be necessary. This study involving human subjects follows previous lab work done by Raman's team demonstrating that combining heart failure medicines slowed the decline of organ function in animals with DMD. The team now plans to start testing the effects of combining heart failure medicines even before signs of heart muscle damage begin to show up.

The research was published in The Lancet Neurology.

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