Researchers at The University of Western Australia have been awarded $490,000 by Duchenne UK to investigate the use of an amino acid commonly used in energy drinks as a treatment for Duchenne Muscular Dystrophy.
Duchenne UK is a charity which aims to fund and accelerate effective treatments and a cure for Duchenne, a rare and fatal muscle wasting disease affecting one in 3500 boys, and very rarely girls, worldwide.
Project partner Save Our Sons contributed $130,000. Save Our Sons is the peak body for Duchenne in Australia, focused on finding a cure for Duchenne by funding research and clinical trials while also raising awareness and supporting the Duchenne community.
Project leader Dr Peter Arthur, senior lecturer in UWA’s School of Molecular Sciences, said the amino acid taurine was found in all animal tissues and was also sold as a health food supplement.
“There is strong data from animal studies to suggest taurine improves muscle strength, reduces inflammation and protects against muscle wasting,” Dr Arthur said.
Emily Crossley, from Duchenne UK, said the funding would enable researchers to better understand whether taurine would work in patients.
“They will then be able to gather information about human urine and blood samples that will be needed to plan a potential clinical trial to look at the safety and efficacy of taurine as a treatment for Duchenne,” Ms Crossley said.
Dr Arthur said there were many advantages to developing taurine as a treatment for Duchenne.
“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,” he said.
“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 Duchenne patients globally, especially where high cost may be a barrier.”
David Stacey (UWA Media Manager) 08 6488 3229/ 0432 637 716