May 2019

Duchenne UK is pleased to announce that the Tamoxifen (TAMDMD) clinical trial is now recruiting in the UK at Alder Hey Children’s Hospital and Leeds Teaching Hospital. Royal Hospital for Children, Glasgow plans to open for recruitment soon.

For information on the trial, visit the DMD Hub website: https://dmdhub.org/trials/tamdmd/ 

• Tamoxifen is a repurposed breast cancer drug also used to treat hormonal disorders in pre-pubescent boys.
• Duchenne UK has committed £1,533,961 to the Tamoxifen trial to date
• The trial will assess the safety and efficacy of Tamoxifen as a potential treatment for Duchenne Muscular Dystrophy (DMD).
• This placebo control, 48-week clinical trial will look at the treatment of Tamoxifen for both ambulant and non-ambulant patients with DMD.

Duchenne UK has worked closely with Professor Dirk Fischer and his team from the University Children’s Hospital in Basel, Switzerland over the last 18 months to co-fund and help get this trial up and running. We have contributed more than £1.5 million towards the Tamoxifen trial. Our initial funding of £575,000 was instrumental in getting the trial started. We are funding the project manager, the UK trials sites and the open label extension study.*

In June 2018, the first patients were dosed in this trial. So far, 31 patients have been dosed from a total of 99 expected participants.

Preliminary data in the DMD mouse model (Dorchies et al. 2013) demonstrated that Tamoxifen reduced fibrosis and increased the thickness of muscle fibres, therefore delaying disease progression.

Tamoxifen has been used to treat breast cancer since the 1980s so has a good safety record. If the trial is successful it could give children with DMD access to a cheap, effective and readily available treatment for DMD in the very near future.

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:

This trial would not be happening in the UK if it wasn’t for the funding and commitment of Duchenne UK. We would like to thank the teams at Leeds, Alder Hey and Glasgow for their commitment to opening as trial sites. We would also very much like to thank the boys and their families who will take part in the trial. 

Repurposing medicines is an important part of our strategy to get treatments to this generation of patients with DMD. Repurposing is an exciting approach to drug development; it involves looking at existing medicines, and testing them to see if they could be effective in treating DMD. This may dramatically reduce the time it can take to get medicines into the clinic to be tested for new indications on patients because these medicines are already approved as safe for use in humans.

The trial is being jointly funded by Duchenne UK, E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy.

Duchenne UK would like to thank Marshall Wace for supporting this project.

We would also like to thank our partner charities and funds for their support of our ongoing work on repurposing: Joining Jack, Alex’s Wish, Duchenne Now, For Felix, Jack’s Mission, Jack’s Aim, Archie’s March, Caring for Connor, Brandon’s Fund, Team Felix, Smile with Shiv, Help Harry, Hope for Harry, Jacobi’s Wish, Jayden's Army, The Smedley Family and Chasing Connor’s Cure .

WE NEED YOUR HELP TO KEEP FUNDING PROMISING RESEARCH, DONATE NOW.

*Funds committed by Duchenne UK to the Tamoxifen Clinical Trial:

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 DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information and interview requests:

Visit www.duchenneuk.org

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