March 2019

-Testosterone is sometimes given to patients with Duchenne Muscular Dystrophy (DMD) who take steroids.

-The £228,562.51 grant from Duchenne UK will fund an extension to the current clinical trial studying the effects of the use of testosterone in DMD patients.   

Steroids are part of the recognised standard of care in treating Duchenne Muscular Dystrophy (DMD). But long-term use of steroids causes a number of side effects including delayed puberty. Testosterone is sometimes given to make boys begin going through puberty.

As the life expectancy of DMD patients increases, more young men are looking to establish relationships and lead independent adult lives. We are funding this research to ensure boys are given the correct hormonal treatment to allow them to develop properly.

Our funds will support the clinical trial at the John Walton Muscular Dystrophy Research Centre (JWMDRC), led by Prof Volker Straub and Dr Claire Wood, treating 15 adolescents with DMD with testosterone to induce puberty. You can read more about the trial here on the DMD HUB website. As well as looking at the effect of testosterone on pubertal development, growth, muscle strength and function, bone mineral density and body composition, the trial is looking at the mood, quality of life and well-being of patients to assess their satisfaction with the benefits of the treatment compared to the side effects.

The initial trial was funded by Duchenne Now.

Emily Crossley, co-founder and co-CEO of Duchenne UK said:

This is an important study that could impact on the quality of life for young men with Duchenne Muscular Dystrophy. We would like to thank our partner charity, Duchenne Now for initiating this valuable project and we are pleased to be able to help fund the next stage.

Emma Hallam, founder of Alex’s Wish said:

Having a son approaching 13 years old who has Duchenne and is on daily steroids, I understand the impact of delayed puberty – especially its impact on muscle strength and bone density.  I know how important it is that we understand the effects of Testosterone and Alex’s Wish fully supports this project. We are so thankful to our supporters for enabling us to invest in such an important project leading us to new treatments that could help improve wellbeing and life expectancy.

The grant, worth £228,562.51 will pay for the extension study of this trial to look at the longer term effects of testosterone treatment following earlier treatment to induce puberty. The extension study will investigate whether testosterone treatment to induce puberty will be enough to support long term testosterone production, or whether further treatment with testosterone is needed for patients on long-term steroids to keep producing testosterone, even if puberty was successfully induced.

If this study can prove that Testosterone for pubertal induction is an effective treatment to address delayed puberty, then we hope to see the use of testosterone included in the standards of care for adolescents with DMD.

Duchenne UK would like to thank our partner charities and family funds for their support of this project:  Alex’s Wish, Caring for Connor, Duchenne Now, Joining Jack, Archie's March, Jacobi’s Wish, Smile with Shiv and Team Felix.

LISTEN TO OUR DECODING DUCHENNE SPECIAL PODCAST EPISODE ABOUT TESTOSTERONE.

Listen on iTunes or below:

Q&A:

What is testosterone?

Testosterone is a sex hormone in both males and females, but is particularly important for the development of an adult male. During puberty, a boy’s testosterone levels increase by up to 30 times as they develop into a man.

Why might testosterone be helpful to people with DMD?

The standards of care for DMD recommend treatment with glucocorticoid steroids. Long term use of steroids almost universally causes delayed puberty by blocking the production of some hormones that regulate puberty. Treatment with testosterone can be used to induce puberty in these patients.

There is some anecdotal evidence it affects penis size, is this true?

If you do not start puberty, then your penis and testicles remain the same size. Inducing puberty increases virilisation – the development from a boy to a man - so taking testosterone will induce this.

What is the aim of the study and this extension?

The study is designed to measure the effect of testosterone treatment on pubertal development, growth, muscle strength, bone health, mood and quality of life and more. This additional funding fully support the extension study, thus helping to understand the long-term effects of puberty induction with testosterone. It will help doctors determine if ongoing testosterone replacement therapy is required and for how long testosterone should be given.

Can taking testosterone interfere with other treatments?

Without steroids, you would have normal pubertal development. There is no reason to exclude testosterone. If you are planning to be enrolled in a specific trial, you must check if taking testosterone is part of the exclusion criteria.

PLEASE TALK TO YOUR DOCTOR BEFORE TAKING ANY MEDICINES

ENDS

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

For more information and interview requests:
Visit www.duchenneuk.org
Molly Hunt – Communications Manager, Duchenne UK 
E: [email protected]