David Stamler, CEO of Alterity Therapeutics, is developing a drug to treat multiple system atrophy (MSA), a rare and rapidly progressing neurodegenerative disease that often presents as Parkinson's disease but is distinct and more aggressive. There is no single genetic cause or specific biomarker, making accurate diagnosis a significant challenge. The lead drug is a novel small molecule designed to manage excess reactive iron in the brain, which drives the disease, and may be effective for other neurodegenerative diseases involving iron dysregulation.

David explains, "Multiple system atrophy is a rare disease, and that's part of the reason people may not know so much about it. It is a neurodegenerative disease, and as the name implies, there are multiple regions of the brain that are affected, hence the term multiple systems that are governed by those regions of the brain. And as the disease progresses, some of these regions degenerate, and you get abnormal function in various areas."

"Now, we like to characterize the disease as a Parkinsonian disorder, which means early on, it can look like Parkinson's disease. And that's kind of a good descriptor to help people understand what it might look like, but it's distinct from Parkinson's disease, and it progresses a lot faster, a lot more rapidly. So it's a disease that people don't know about, probably because no one famous has been diagnosed with MSA, although I'm sure various famous people have probably had the disease and maybe didn't know it."

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Drug Targets Iron Dysregulation in Rare Neurodegenerative Disease Multiple System Atrophy with David Stamler Alterity Therapeutics TRANSCRIPT