Researchers at The University of Western Australia (UWA) have been awarded close to $490,000 from two organizations to explore the potential use of an amino acid called taurine as a treatment for Duchenne muscular dystrophy (DMD).
Duchenne UK granted £273,648 (approximately $356,515) to support the study, while Save our Sons Duchenne Foundation, an Australian DMD patient organization, has kicked in an additional AUS$183,000 (about $130,000).
Taurine is a natural amino acid that can be found in all animal tissues, and is also often sold and used as food supplement. While the safety of taurine has been widely explored in more than 30 clinical trials, its therapeutic potential for DMD patients is not yet fully understood.
Led by Peter Arthur, PhD, who is a senior lecturer at UWA’s School of Molecular Sciences, the team will use the funds to conduct preclinical studies in mouse models of DMD to further understand the potential benefits of taurine treatment.
The collected data is expected to provide evidence of the safety and therapeutic potential of this alternative strategy to support the launch of future taurine clinical studies in human DMD patients.
“Taurine looks very promising. So, we are very grateful for the [Duchenne UK’s] 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,” Arthur said in a press release. “If we get good results, we will be able to trial taurine in DMD patients.”
Taurine is administrated orally, representing an advantage over injectable therapies to patients and caregivers. It is also cheap and very stable, potentially overcoming one of the major limitations of DMD therapies, which are, in most cases, very expensive.
Results from a previous pilot study revealed that by increasing taurine consumption or production, it was possible to improve several aspects of muscle function, reduce inflammation, and protect against muscle wasting in mice lacking the dystrophin protein — the hallmark of DMD.
“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,” said Klair Bayley, executive officer of clinical care and advocacy at Save Our Sons Duchenne Foundation. “With every passing day muscles progressively get weaker for those with Duchenne, so we have no time to waste in finding treatments.”
Duchenne UK’s Innovate Grant aims to support projects focused on finding new therapeutic strategies for DMD, in particular those that are close — within a two- to five-year time frame — or ready to enter clinical trials.