This is a project which will investigate the use of four drugs currently being used to treat cancer as potential treatments for Duchenne.

The project is being led by Professor Winder in the Department of Biomedical Science at the University of Sheffield. The idea behind this research stems from observing some of the similarities between cancer and Duchenne whereby ‘faulty switches’ lead to damage to the body. It is suggested that the same drugs that are used to treat cancer could be used to treat Duchenne. So far two of the compounds have been tested in mdx mice, with encouraging results. The next step is to use all four compounds individually and in combination in mouse models, over a longer period of time. Because the drugs used in this project are already approved for cancer patients they could potentially be fast tracked to the clinic for Duchenne patients.

Researchers at the National Institutes of Health’s National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) revealed that a drug called SU9516, originally developed to treat leukemia, has been shown to be effective in improving muscle function and slowing indicators of disease progression in mouse models of DMD.  Integrin is a protein which stabilizes muscle and stimulates regeneration. Rather than targeting mutations, SU9516 aims to enhance muscle repair through increasing integrin production. Plans are underway to develop a safer version of the drug to potentially offer a treatment for Duchenne patients.