Promising drug news for muscular dystrophy patients; but no breakthrough, warn researchers

Erectile dysfunction and pulmonary hypertension medicine can help re-energize blood flow for those suffering from Duchenne muscular dystrophy, according to a new study published in Neurology.

According to the study the drugs restored blood flow to muscles starved of oxygen, which is what happens with patients suffering from DMD. But researchers warn the study results are not a huge breakthrough.

The study, conducted by researchers at the Cedars-Sinai Heart Institute, say the drugs reversed defective blood vessel mechanisms inherent in the fatal disease that kills most patients by their 30s.

"The effects were immediate and dramatic, raising the question: If a single dose restores blood flow to muscle while the drug is in the patient's system, could ongoing tadalafil administration provide sustained benefits, possibly preserve muscle and slow disease progression? If so, this would offer a new therapeutic strategy for DMD, and we have launched a randomized Phase III clinical trial to find out," said senior study author Ronald Victor, MD, director of the Cedars-Sinai Center for Hypertension, associate director of clinical research at the Heart Institute and the Burns and Allen Chair in Cardiology Research.

In previous studies, in 2012, Victor and his research team found that Tadalafil, which is also known as Cialis, proved to fully restore blood flow in patients dealing with Becker muscular dystrophy.

"Steroids and cardiac-protective blood pressure medication are increasingly prescribed at early ages for patients with Duchenne muscular dystrophy in an effort to delay by a few years the most devastating effects of the disease. But these treatments have no effect on the blood vessel dysfunction that prevents muscles from getting the oxygen they need," said Victor. "In contrast, in our study, a single dose of tadalafil or sildenafil had an immediate effect. These are well-studied, well-tolerated drugs that are already on the market. If additional study confirms their benefits, repurposing the drugs for muscular dystrophy patients could quickly transform clinical practice."

Currently there is no cure or long-term treatment for DMD which affects one in every 3,5000 male babies.

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