Access to treatments

First patients dosed in charity funded clinical trial, to test the safety and effectiveness of tamoxifen for DMD

Duchenne UK invests £575,000 to support the study.

Most people associate the medicine tamoxifen with treating certain types of breast cancer. But a clinical trial has begun this week to test it as a potential treatment for Duchenne muscular dystrophy.

We are delighted to share the news that the first two patients were dosed yesterday, June 26th, in the TAM-DMD study. The trial started at the University Children’s Hospital Basel, in Switzerland under Prof Dirk Fisher M.D, Senior Physician, Neuro and developmental Paediatrics.

Duchenne UK has worked closely with the team in Switzerland (pictured below) for the last 12 months to help get this trial up and running, and has committed £575,000, along with E-Rare, Duchenne Parent Project in Holland, and the Monaco Association against Muscular Dystrophy.

Tamoxifen has been used to treat breast cancer since the 1980s. It is also used for hormonal disorders in pre-pubescent boys.

This placebo control, 48-week clinical trial will look at how safe and effective Tamoxifen might be as a potential treatment for DMD.

We are pleased to share the news that the trial will be for both ambulant and non-ambulant patients with DMD.

Professor Dirk Fischer, from University Children’s Hospital in Basel, Switzerland 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.”

This trial will be running in the UK, at Leeds and Alder Hey.

To keep up to date with developments, please sign up to the HUB newsletter.

Duchenne UK is contributing £575,000 to the study and will fund one European trial site and two in the UK: Alder Hey and Leeds, along with the project manager for the study, based in Switzerland.

Duchenne UK is working through 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 clinical trial.

A study of Tamoxifen in the DMD mouse model, (Dorchies et al. 2013), showed that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.

(Read our Tamoxifen Factsheet on Tamoxifen here)

Because Tamoxifen is already used as a treatment in other disease areas, it is a cheap and readily available medicine. It also has a good safety record in adults. Duchenne UK is committed to developing existing medicines as potential treatments for DMD. This approach is known as repurposing. 

The trial marks an important milestone in Duchenne UK’s commitment to developing existing medicines as potential treatments for DMD. The advantage to 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. They could offer a potentially quicker path to the clinic and approval.

Alex Johnson and Emily Crossley, co-founders of Duchenne UK said:

“This milestone is a very exciting moment for Duchenne UK. As well as funding the TAMDMD trial and project manager, we have also worked with the sites in the UK to ensure they are able to participate in the trial. We have done this through the DMD Hub by funding the doctors, nurses and support staff to run the trial as well as providing training, mentoring and resources.”

For more information and updates on the trial visit: dmdhub.org/trials/tamdmd/ 

The TAMDMD 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 their generous grant in support of this project. We would also like to thank our partner charities, Joining Jack and Alex’s Wish, 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.

Published on 27 June 2018

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Note 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

Visit www.duchenneuk.org

Interview requests:

Molly Hunt – Communications Manger, Duchenne UK 

E: [email protected]