Tadalafil Drug Raises Hopes for Boys



Tadalafil, also known as Cialis and Adcirca, is a drug designed to increase blood pressure in the lungs and help erectile dysfunction (ED). Although it is not a cure for ED, tadalafil has raised the hope for those suffering from Duchenne muscular dystrophy (DMD), a fatal genetic condition that affects boys.

Duchenne muscular dystrophy is a disorder inherited from the mother that cripples boys quickly and severely. With genetic testing, DMD can be detected during pregnancy with a 95 percent level of accuracy, appearing once out of every 3,600 male infants. To this date, it has no known cure. Girls may carry and pass on the defective gene without showing any symptoms. Boys with DMD develop weakness mostly in their legs and pelvis, compromising their basic motor skills. On top of all of these issues, the child can develop enlarged muscles that are replaced with fat, deformities, heart failure, loss of muscle mass and respiratory issues. Boys begin to show signs as early as infancy up until the age of 6, often losing the ability to walk by the age of twelve. Treatment may delay the effects in boys, but it does nothing to slow the eventual death that occurs from breathing complications, occurring roughly around the age of twenty-five.

Steroids have been known to slow the progression of the disease, and in a recent case study, tadalafil has improved and even restored some of the damage done by the inherited disorder, raising the hopeful possibility of an improved quality of life for the affected boys. Ronald Victor, director of the Cedars-Sinai Center for Hypertension, believes the drug can lead to improved and innovative therapies for children with DMD. The studies showed that patients with DMD have impaired blood vessel function, creating an abnormal blood flow. Tadalafil works by effectively and immediately raising the blood pressure, “correct[ing] defective blood vessel mechanisms and restor[ing] blood flow to exercising muscles,” all from a single dose.

There are 22 sites in the United States and over 30 sites around the world dedicated to the study of the effects of the drug on DMD patients. Eli Lilly and Company and Sanofi are working to get the drug licensed and approved for over-the-counter sales. Today, the drug is only available through prescription. Based on having recorded over $14 billion in sales since its launch, tadalafil will most likely be a huge success if it is approved. Hoping to discourage any rising concerns, Lilly and Sanofi are currently recruiting participants for its phase three trial of tadalafil, in order to assure the safety of its use for men and search for any side effects for boys. If everything goes according to plan, Sanofi hopes to exclusively sell the drug over the counter in Europe, Canada, Australia and the United States. Regardless of whether tadalafill can actually slow the progression of the disease or protect dystrophic skeletal muscles in boys, many parents who have raised boys with DMD can remain hopeful for more scientific breakthroughs such as these as the disease continues to carry on into the next generation.

By Sophia Bien

Medline Plus
Drug Discover & Development
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Clinical Trials