AI-guided screening identifies nine drugs that may slow childhood dementia
An Australian research collaboration has identified several existing TGA-approved medicines that could help slow brain damage in children with a rare form of dementia—offering hope for faster treatment options.
An Australian research team has discovered several existing, TGA-approved medications that could potentially slow brain damage in children with Sanfilippo syndrome, a rare form of childhood dementia with no widely available treatments. By utilizing patient-derived brain cells grown in a lab, alongside advanced imaging and artificial intelligence, the researchers rapidly tested the approved drugs to identify those that improve brain cell health.
The study, published in Nature Communications, focused on Sanfilippo syndrome, a devastating genetic disorder that affects 1 in 2,900 children in Australia, with half dying by age 10. Researchers reprogrammed skin cells from affected children into brain cells that replicate the disease's progression, revealing toxic buildup, inflammation, and ongoing cell loss.
The team screened 63 existing medicines and found nine that significantly improved cell function within two weeks, with some reducing brain cell damage and others restoring critical signaling linked to learning and behavior. Combining treatments may lead to stronger, longer-lasting effects. Lead researcher Professor Cedric Bardy from Flinders University emphasizes that this approach could expedite treatment discovery by using human brain cell models combined with machine learning.
The findings offer hope for managing daily symptoms and slowing irreversible brain damage in children until a cure is discovered.
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