Drug boost for muscular dystrophy

“Early treatment with a drug can delay the onset and progression of heart failure in children with Duchenne muscular dystrophy, research suggests.
DMD is an incurable genetic disease causing muscle wastage, which often leads to fatal cardiac problems.
The study shows the drug, perindopril, can slow heart muscle degeneration – and thus ward off heart failure.
The research by Paris’s Cochin Hospital involved 57 children, says the Journal of the American College of Cardiology
Lead researcher Professor Denis Duboc said: “For the first time, we have shown that it is possible to slow progression in this rare degenerative disease.
“In DMD, the heart muscles are affected and cardiac problems are fatal in around 40% of children.”
The five-year study focused on the effect on perindopril, a drug from a class known as ACE inhibitors, widely used to treat high blood pressure and heart failure.
Some 57 children with DMD received either perindopril, or a dummy drug.
Eight in the dummy group went on to develop signs of heart failure, and three died from the condition.
In contrast, just one of the perindopril group showed signs of heart failure, and none died from the condition during the study.
Professor Duboc said the results suggested early treatment with perindopril might also benefit other people genetically predisposed to heart failure.
DMD, one of the most common forms of muscular dystrophy, is caused by a lack of a protein called dystrophin which helps keep the muscles intact.
It strikes children at a young age, and affects almost exclusively boys who rarely survive beyond their early 30s.
Findings welcomed
Professor Francesco Muntoni, an expert on paediatric neurology at Imperial College London, told the BBC News website it made sense that drugs found to be effective at treating other forms of heart failure should work for DMD patients as well.
“Interestingly, the treatment was effective in preventing the development of the cardiomyopathy, suggesting that it should be started as early as indicated in these children, before significant damage has occurred,” he said.
“Further work will be needed to assess the effect of this treatment on patients who have already developed a significant cardiomyopathy.”
“This is an important area of research also because life expectancy in Duchenne has greatly improved following the initiation of non-invasive ventilation in the last decade of life, extending life of another 10 years or so.
“This, however, increases the potential for patients to develop a cardiomyopathy.” Professor Muntoni said it was also possible that the drug could benefit people with other forms of muscular dystrophy in which heart failure was a potential factor.