New 'cocktail' drug could benefit up to 45 per cent of patients with Duchenne muscular dystrophy

The U.S Food and Drug Administration has already approved other similar treatments, including viltolarsen, which is based on Yokota's research. Each, however, has limited applicability. This new "cocktail" treatment could help many more patients.

"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," said Yokota, who is also the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair.

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

The research was published this week in the Proceedings of the National Academy of Sciences.

Yokota's team tested the new synthetic drug in patient-derived muscle tissue in test tubes and in mice. They found signs of dystrophin production, muscle building and improved heart function.

DMD often leads to extreme skeletal body weakness, yet most patients actually die from heart failure. Existing exon-skipping treatments do not penetrate the heart muscle - a limitation this new cocktail addresses, according to Yokota.

"Our cocktail combines the antisense oligonucleotides with a new peptide which allows the drug to penetrate the heart muscle," he said.

The cocktail still needs to undertake toxicology testing and go through the regulatory steps to conduct clinical trials. Yokota and his colleagues recently launched a company to help commercialize the drug.

The full article can be found here.

10.1073/pnas.2112546119