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Clinical trial first to test heart drug regimen for Duchenne muscular dystrophy
Date:6/26/2012

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The image analysis includes an overlay tool that divides the heart into 24 equal sub-segments. As the heart beats, the segment grids move in line with the region of the heart they cover. The grids start out as perfectly square, but then contort in conjunction with a twisting motion the heart makes to force blood through the body. This allows physicians to measure heart function region by region to catch the smallest of declines.

"On an individual basis, measuring each of the grids doesn't tell you very much," Hor explained. "But when you add up the grids, you can see cumulative changes and catch small but important declines in heart function. We believe this test will help us pick the best time to begin drug treatment."

Preclinical drug tests

The decision to test the drug eplerenone is based in part on extensive clinical studies of DMD patients at Cincinnati Children's and in mouse models of DMD at OSU spear led by Dr. Hor's collaborator Dr. Subha Raman. Another factor is its success in treating people with certain types of advanced heart disease.

The drug is an aldosterone antagonist, a diuretic that helps the body get rid of excess fluid (tissue swelling) while retaining sufficient potassium levels. It blocks the buildup of fibrotic material and scarring that over time can turn heart muscle into non-functioning fatty tissue.

The drug's success in treating fibrosis in adult heart patients has led to it being sought for off-label treatment in patients with DMD, creating a sense of urgency behind the current clinical trial, Hor said.

In the mouse studies, early treatment with eplerenone resulted in reduced heart fibrosis and improved cardiac function. Researchers treated two groups of mice, one at 8 weeks of age and a second at 4 weeks. As the mice aged, the researchers noted normal LVEF and functional improvement in both groups, although reported the benefit was more pronounced
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Contact: Nick Miller
nicholas.miller@cchmc.org
513-803-6035
Cincinnati Children's Hospital Medical Center
Source:Eurekalert

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