Posted: 27 November 2024
Associate Professor Shiang (Max) Lim, of SVI’s O’Brien Department, has been awarded funding from the Friedreich’s Ataxia Research Alliance (FARA) to better understand the effect of omaveloxolone – a recently FDA-approved drug – on heart disease in people with Friedreich ataxia.
Friedreich ataxia is a rare and progressive neuromuscular disorder that primarily affects the nervous system, muscular system, and the heart. There is currently no cure for Friedreich Ataxia, and treatment has been historically focused on managing symptoms and providing supportive care, such as physical therapy, speech therapy, and mobility aids.
The recent Food and Drug Administration and European Commission approvals of SKYCLARYS ® – omaveloxolone – to treat Friedreich ataxia in individuals aged 16 and older is a significant step forward for the Friedreich ataxia community.
“Omaveloxolone is a drug that activates a protein that helps the body fight off harmful substances,” said Associate Professor Max Lim.
“It shows promise in reducing neurological symptoms compared to placebo and slowing down the progression of the disease, but we still don’t know much about how it affects heart disease in Friedreich ataxia. This project aims to fill in this important knowledge gap.”
Associate Professor Lim and his team want to find out exactly how omaveloxolone affects the cells in the hearts of people with Friedreich ataxia. To do this, they will be studying heart cells grown from stem cells of individuals with Friedreich ataxia.
They will also use a 3D model of a Friedreich ataxia heart in a dish, which includes different types of cells found in the heart, to see how omaveloxolone might indirectly affect heart cells through interactions with nearby non-heart cells.
Additionally, they will also test how omaveloxolone works with other experimental drugs currently being tested for Friedreich ataxia. Their goal is to gather important information on the effect of omaveloxoloneon the heart disease in people with Friedreich ataxia.
Lim has been awarded more than $250,000 (USD), the Keith Michael Andrus Cardiac Research Awardfrom the Friedreich Ataxia Research Alliance (FARA).
