Erectile dysfunction drugs could be an effective treatment for children suffering from muscular dystrophy.
The common drug could help restore blood flow to the oxygen-starved muscles of boys who have the condition, a Cedars-Sinai Medical Center news release reported.
The muscle-wasting disease is much more prevalent in males; it affects one in every 3,500 male babies and is the most common disease in children.
In muscular dystrophy cases muscle weakness occurs in early childhood and often leads to deformities of the "arms, legs, and spine," the news release reported. The heart and respiratory muscles can fail by the early teen years.
Researchers looked at a case study of 10 boys suffering from Duchenne muscular dystrophy (DMD). They found a single dose of a drug commonly prescribed for erectile dysfunction or hypertension helped restore blood to exercised muscles.
"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," 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, said in the news release.
The condition was caused by a genetic defect that eliminates a protein called dystrophin in themembranes of muscles cells.
"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," Victor said. "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."
