Many parents report using supplements and nutraceuticals for their sons. However very little evidence exists on whether these supplements are safe and effective, and what the right dose should be. Supplements are often expensive and some can cause harm. Duchenne UK has invested more than half a million pounds in investigating nutraceuticals and compounds that derive from nutraceuticals. Read more here.

Today we are pleased to announce a grant of £273,648 to the University of Western Australia to investigate the use of taurine as a treatment for Duchenne muscular dystrophy (DMD). We are pleased to partnering with Save our Sons Duchenne Foundation, the Australian DMD Patient Organisation on this project. They are contributing AUS$65,000.  

Taurine is an amino acid that is found in all animal tissues. It can also be bought as a health food supplement. There is strong pre-clinical data to suggest taurine improves muscle strength, reduces inflammation and protects against muscle wasting in the DMD (mdx) mouse model.

This project will perform further animal experiments to understand better how taurine would work in patients.

The project will then gather information on human urine and blood samples that would be needed to plan a potential clinical trial to look at the safety and efficacy of Taurine as a treatment for DMD.

Developing taurine as a treatment for DMD has a number of attractions:

The safety and pharmacokinetics of taurine in humans is already well understood and very few adverse side effects have been reported in more than 30 clinical studies. Taurine can be taken orally, which is much more pleasant and less of a burden on patients and caregivers than injections. And it’s cheap and stable. This gives taurine the potential to be widely available to many DMD patients globally, especially where high cost may be a barrier. 

The project has been reviewed by TACT and the protocols for the project have been designed to reflect recommendations from the TACT review. If taurine is shown in this study to have benefit when treating DMD, the next stage would be to plan a clinical study in DMD patients.  

Emily Crossley from Duchenne UK said: “We launched the innovate grant call last year and required all applicants to attend TACT and receive a formal review. Our Research Director Dr David Bull has worked very closely with the UWA group to fine tune the processes and help ensure the project produces results of value and application.”

Dr Peter Arthur, Senior Lecturer, School of Molecular Sciences, The University of Western Australia said: “Taurine looks very promising. So, we are very grateful for the Innovate Grant as it will allow us to undertake rigorous testing in an animal model of dystrophy and develop the tools we need to monitor the effectiveness of taurine treatment. If we get good results, we will be able to trial taurine in DMD patients.”

Klair Bayley from Save Our Sons Duchenne Foundation said: “We are thrilled to be supporting Australian research that could be rapidly translated into a cheap and readily available treatment to help keep muscles stronger for longer for those living with Duchenne. With every passing day muscles progressively get weaker for those with Duchenne, so we have no time to waste in finding treatments.”

The Innovate Grant call invited proposals for funding projects up to £1million, dedicated to finding new treatments for DMD. The grant call aims to identify drug development projects that are ready for trial, with compelling pre-clinical data, including data in animals, or have the potential to be in clinical trials within a 2 – 5 year timeframe. Shortlisted applicants are required to complete a TACT application.

Duchenne UK has an impressive track record in supporting research aimed at accelerating effective treatments for DMD.  Supporting and accelerating innovative research is at the heart of what we do at Duchenne UK. 

We have put together a short Q&A to explain why we are excited about this project and what it could mean for the Duchenne community. 

Q: What is so exciting about this project?

A: There is already significant pre-clinical work suggesting taurine improves muscle strength, reduces inflammation and projects against muscle wasting in DMD mice. This project will build on that pre-clinical work to create a stronger evidence base for taurine as a treatment for DMD.

Q:If this project is successful, what will the next stage be?

A: If taurine is shown in this study to have benefit when treating the DMD rat model, the next stage would be to plan a clinical study in patients with Duchenne muscular dystrophy. The project has been designed carefully to gather the information we will need to plan the trial.

Q: What is the appeal of using Taurine as a treatment for DMD?

A: Taurine is an amino acid which occurs naturally in all animal tissues and can be bought as a health food supplement. The safety of taurine and how the body handles it is already well understood. Also, taurine is cheap, readily available and stable. This means taurine could potentially be a promising treatment for people with DMD all over the world, especially in areas where high cost may be a barrier.

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