Clinical trial aims to find curative treatment for MSA

25 Oct 2022

Multiple System Atrophy (MSA) is a rare degenerative neurological disorder affecting approximately 1,000 Australians. It presents similarly to Parkinson’s disease, but is rarer, progresses more quickly and is less responsive to treatments. Currently there is no effective treatment for MSA, with patients experiencing progressive deterioration and significantly reduced life expectancy.

In the hope of changing the poor prognosis for MSA patients, St Vincent’s is participating in a Phase 2, international, multi-centre Clinical Trial that hopes to find an effective treatment. As the first clinical trial site in Australia, the randomised, double-blinded, placebo controlled study will explore the effect of a new drug ATH434, which is designed to disrupt the accumulation of iron and a protein alphasynuclein associated with the neurodegenerative process.

Patients radomised to the active arm of the study will receive ATH434 for 12 months, enabling the researchers to detect changes and ultimately assess efficacy of the drug and the relative impact on patient outcomes.

“The thing that excites me about this study is the hope that it brings”, Dr Stephen Tisch, St Vincent’s Neurologist and Research Lead.

“It’s bringing a level of hope for patients who have always been told there is nothing that can be done. While there are treatments for symptoms, they don’t change the course of the disease for which there is currently no cure”.

“The discovery of effective disease modifying treatment for MSA would represent a major breakthrough in what is otherwise a devastating and incurable condition.”

Recruitment for participants is currently underway.

Dr Stephen Tisch