AI finds existing drugs that may slow childhood dementia

Australian researchers used AI to identify existing medicines that may slow Sanfilippo syndrome, offering hope for faster treatment options for childhood dementia. The study published in Nature Communications used patient-derived brain cells to identify nine of 63 approved drugs that improved cell function within nine drugs, including a combination of repurposed drugs that can return brain cell activity to healthy levels. The study focused on Sanfilippo syndrome, a rare childhood dementia that causes progressive loss of memory, behavior and physical abilities. There are currently no widely available treatments. The team reprogrammed skin cells from affected children into brain cells that mimic the disease, reproducing toxic buildup, inflammation and cell loss. The findings suggest combining treatments may produce stronger, longer-lasting effects, and because the drugs are already approved, they could move into clinical trials much faster than entirely new therapies.