Erectile Dysfunction Drug Shows Promise As Muscular Dystrophy Treatment
Researchers from Cedars-Sinai Heart Institute recently found that tadalafil, a drug commonly prescribed to treat erectile dysfunction or pulmonary hypertension, improved blood flow in the muscles of young patients with Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a progressive and fatal disease primarily affecting young men and boys. Duchenne muscular dystrophy causes patients to lose muscle function, and there is currently no disease- specific treatment in existence. Typical treatment includes steroid medications, which can slow down the disease’s progression, but these medications are not often well tolerated.
The researchers at Cedars-Sinai started the study by testing 10 boys between the ages of 8 and 13 with Duchenee muscular dystrophy who were being treated with corticosteroids. The researchers tested the blow flow to the patients’ muscles when resting and when completing a handgrip exercise, then compared the results to 10 healthy boys of similar age. The boys with muscular dystrophy were shown to have blood flow abnormalities. Next, the researchers conducted the same test, but after the boys with muscular dystrophy received treatment with tadalafil. The researchers waited two weeks to conduct the 2nd test, which showed no difference in blood flow response between the healthy boys and the boys with muscular dystrophy.
Ronald G. Victor, who led the study and is the assistant director of the Cedars-Sinai Heart Institute, said that the effects of the study were “dramatic” and that at higher doses, the results were even more pronounced. He continued by saying that the results did not indicate a cure, but a step “toward identifying potential treatments.”
Victor says, "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."