Action Duchenne, Alex’s Wish, Duchenne Now and Harrison’s Fund are  co-funding a research project.  Called Repurposed Cancer Therapeutics as Treatments for DMD, the project will be led by Professor Steve Winder in the Department of Biomedical Science at the University of Sheffield.

With a total cost of £120, 935, this project hits the strategic aims of each of the funding charities by showing the potential to treat all Duchenne patients and the possibility of a fast track to the clinic.

The project will investigate the use of four drugs that are currently used to treat cancer as potential treatments for Duchenne, using mouse models. The project aims to assess:

1.    the long-term efficacy of three tyrosine kinase inhibitors in reducing the dystrophic patho-physiology in mdx mice.

2.    the long-term efficacy of a proteasome inhibitor in reducing the dystrophic patho-physiology in mdx mice.

3.    the possible synergistic effect of tyrosine inhibitors combined with proteasome inhibitors in further reducing the dystrophic patho-physiology in mdx mice.

In people living with Duchenne Muscular Dystrophy, a faulty signal in the cell acts as a switch which leads to disruption of the muscle cell surface.  This ‘faulty switch’ is part of the cause of Duchenne.  If this ‘faulty switch’ could be turned off it could stop or reduce the muscle damage associated with DMD. Some cancers are caused by the same kind of faulty switch, and therefore the same drugs that are used to treat these cancers may also have therapeutic benefit in Duchenne.

The team behind this project have previously experimented with these drugs in zebrafish models that have Duchenne, where they worked as expected.  They have also used two of the compounds on 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.  If successful this project will be a crucial step in gaining approval for a clinical trial of these treatments in human subjects.  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 if this research project is successful.