Duchenne UK is delighted to announce that it 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 £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. Duchenne UK is working with the DMD Hub to support the trial sites in the UK for the Tamoxifen study. The UK sites will be Alder Hey Children’s Hospital in Liverpool and Leeds Children’s Hospital. Duchenne UK, along with Alex’s Wish and Joining Jack, are also providing funding to support clinical staff at Alder Hey and Leeds to deliver the trial.

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 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."

Emily Crossley & Alex Johnson, 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."

The trial marks an important milestone in Duchenne UK’s 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.

Duchenne UK would like to thank its partner charities, Joining Jack, 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, Jayden's Army and Chasing Connor’s Cure, for their support of our ongoing work on repurposing.

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NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein

dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct

  • Individual Donation – Donate

  • If you are a family or friend affected by DMD you can set up your own fund

    with Duchenne UK – Family and Friend Funds

  • Take part in one of our fundraising events – Events

  • Text DUCH10 £10 to 70070

For more information and interview requests:

Visit www.duchenneuk.org

Molly Hunt – Communications Manager, Duchenne UK E: [email protected]