Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy

Public release date: 26-Jun-2012 [ | E-mail | Share ]

Contact: Nick Miller nicholas.miller@cchmc.org 513-803-6035 Cincinnati Children's Hospital Medical Center

CINCINNATI The first landmark randomized clinical trial for a cardiac drug regimen in Duchenne muscular dystrophy (DMD) is testing whether earlier treatment can stop or slow down heart damage that usually kills people with the disease.

The study is a collaboration of Cincinnati Children's Hospital Medical Center, Ohio State University (OSU) and The Christ Hospital in Cincinnati.

Extensive research including studies in mouse models of DMD suggests an anti-fibrosis drug long used to treat heart attack victims, eplerenone, could help people with the disease, said Kan Hor, MD, a principal investigator and a physician/researcher at Cincinnati Children's.

Disease-associated heart damage is the leading cause of death in patients between ages 20 and 30 the maximum life span for people with DMD.

A key element of the new treatment approach is using enhanced cardiac magnetic resonance imaging, Hor said. The technique more sensitive than standard echocardiogram tests now used in DMD is designed to catch minute declines in heart function much earlier in a child's life.

"Standard diagnostic techniques rarely catch significant heart damage in DMD in the first decade, although research shows scar tissue is already developing in many patients before it's clinically detectable through current methods," said Hor, who works in the Cincinnati Children's Heart Institute. "We want to determine the optimal time point to detect these early changes in heart function and start cardiac therapy with anti-fibrotic medication."

A genetic disease that affects mostly boys, DMD is the most common form of muscular dystrophy. Most children begin losing the ability to walk between the ages of 7 and 13. With decades of research, testing and increased emphasis on multidisciplinary care, physicians have been able to use steroids and other drugs to preserve some ambulation, pulmonary function and push survival ages well into the 20s.

Still, there remains no consensus on how to best manage DMD-associated heart fibrosis and disease, Hor said. Often, between the ages of 7 and 10, echocardiogram results

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Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy