Our Partner Charity Duchenne UK is funding a clinical trial to assess the safety and efficacy of Tamoxifen as a potential treatment for Duchenne Muscular Dystrophy.
Duchenne UK is contributing £400,000 to the study and will fund one European trial site and two in the UK, along with a project manager for the study, based in Switzerland. Duchenne UK is working with the DMD Hub to support the trial sites in the UK for the Tamoxifen study.
Tamoxifen has been used to treat breast cancer since the 1980s. It is also used for hormonal disorders in pre-pubescent boys. Preliminary data demonstrated that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.
Along with Duchenne UK and Joining Jack, we are specifically providing support to fund three children to access the clinical trials of Tamoxifen in the UK. This is fabulous news for us as funding these trials will not only impact directly those children taking part, meaning they will get early access to Tamoxifen, but will potentially increase the speed to approval of these drugs.
Working with Duchenne UK, means that we join them in their commitment to develop existing medicines as potential treatments for DMD, an approach known as repurposing. The advantage of this approach is that these medicines have already been tested for safety in humans and so their development time has the potential to be cut dramatically and