Tamoxifen Trial

JOINING Jack is delighted to announce that alongside Duchenne UK it is co-funding a clinical trial to assess the safety and efficacy of Tamoxifen as a potential treatment for Duchenne Muscular Dystrophy.

Joining Jack is contributing £291,000 of a total £575,000 to the study and will fund one European trial site and two in the UK, along with the project manager for the study, based in Switzerland. Joining Jack is working with the DMD Hub to support the trial sites in the UK for the Tamoxifen study. The sites will be at Alder Hey Children’s Hospital in Liverpool and at Leeds Children’s Hospital. Along with Duchenne UK and Alex’s Wish, Joining Jack, are also providing funding to support clinical staff at Alder Hey and Leeds to deliver the trial.

The clinical trial will assess the safety and efficacy of Tamoxifen as a potential treatment for Duchenne Muscular Dystrophy. The trial marks an important milestone in our 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 they could offer a potentially quicker path to the clinic and approval.

Tamoxifen has been used to treat breast cancer since the 1980s. It is also used for hormonal disorders in pre-pubescent boys. Preliminary data in the DMD mouse model (Dorchies et al. 2013) demonstrated that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.

The trial is being jointly funded by Joining Jack, Duchenne UK, E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy. The trial is due to begin in the 4th quarter of 2017.

Professor Dirk Fischer, from UKBB, the University Children’s Hospital in Basel, Switzerland in Switzerland who is running the study, said: “Duchenne UK and the other patient organisations came in with funding at a very critical moment. Despite a positive evaluation and a recommendation by the E-Rare review process, this trial would not have been possible at all without the co-funding from Duchenne UK and the other patient organisations. I am extremely grateful for their ongoing support, which has included not only financial help, but also help negotiating with regulatory authorities and other involved parties."

Alex Johnson and Emily Crossley, CEOs and Co-founders of Duchenne UK said: "We are delighted to announce our funding and support for the Tamoxifen trial, and our support for the trial sites in the UK who will be running the study. Tamoxifen is a cheap and readily available medicine, with a good safety record in adults. We are keen to test this in patients with Duchenne Muscular Dystrophy. If this trial is successful, we could see a cheap, effective and readily available treatment for Duchenne Muscular Dystrophy in the very near future."

Duchenne UK and Joining Jack would like to thank its partner charities, Alex’s Wish, For Felix and Jack’s Mission for supporting this study. We would also like to thank Caring for Connor, Brandon’s Fund, Team Felix, Smile with Shiv, Jack’s Mission, Help for Harry and Chasing Connor’s Cure, for their support of our ongoing work on repurposing.