There are currently no treatments available for multiple system atrophy (MSA), a rare and fatal neurodegenerative disorder caused by the accumulation of alpha-synuclein in the brain. MSA causes problems with movement, balance, and the control of some involuntary bodily functions such as blood pressure and bowel and bladder function. Last month, Alterity Therapeutics published promising results in Movement Disorders of ATH434 in a mouse model of the disease.
ATH434 is a small molecule that targets alpha-synuclein in MSA and Parkinson’s disease (PD). In the study, researchers demonstrated that it not only protected neurons from the damaging effects of alpha-synuclein, but that it also improved motor function—an important finding given that problems with movement are a significant source of disability in patients with MSA.
Alterity has completed a study of ATH434 in a trial of healthy volunteers and is on track to begin a Phase 2 trial in MSA later this year.