'Cocktail' drug developed at U of A to treat neuromuscular disease

A new "cocktail” drug to help lessen the symptoms of patients with Duchene muscular dystrophy (DMD) is being developed at the University of Alberta.

The concoction is a mixture of six treatments and is now being tested for efficacy, according to the lead researcher Toshifumi Yokota.

DMD, or chronic muscle-wasting disease, affects six of every 100,000 people, usually boys, the study said. And it’s the most common inherited neuromuscular disease.

There are various mutations of DMD in the body’s largest gene, dystrophin. Researchers note dystrophin has 79 sections, and if even one is missing, the body cannot produce the protein and the muscles begin to degenerate.

There is currently no cure for DMD, but there is a new class of drugs that can act as a “band-aid.”

“Each of the previously developed exon-skipping molecules has been able to treat only around 10 per cent of DMD patients because they have different mutations to their exons in different locations within the gene,” Yokota explained.

”Our approach is to skip over 11 exons all at once, which would allow us to treat approximately 45 per cent of patients.”

Yokota said his team has tested the new synthetic drug in “patient-derived” muscle tissues in test tubes and mice.

Researchers also discovered signs of dystrophin production, which include muscle regeneration and improved heart function.

According to Yokota, this finding is important because DMD often leads to extreme body weakness and heart failure.

“Existing exon-skipping treatments do not penetrate the heart muscle - a limitation this new cocktail addresses,” he said.

At this point the "cocktail" still needs to undergo toxicology testing and clinical trials.

However, according to the study, Yokota and his colleagues have already launched a company to help comercialize the drug.

To read the full study, click here.