AI screening finds nine existing drugs that may slow childhood dementia

Australian researchers have identified nine existing TGA-approved drugs that may slow brain damage in children with Sanfilippo syndrome, a rare childhood dementia, offering hope for faster treatment options. The study, published in Nature Communications, used lab-grown brain cells from affected children combined with artificial intelligence to screen 63 approved medicines, finding nine that improved cell function within two weeks. Several drugs reduced brain damage, while others restored signaling linked to learning and behavior. Sanfilippo syndrome is one of over 100 genetic disorders affecting 1 in 2,900 Australian children, with half of all childhood dementia patients dying by age 10. Because the drugs are already approved, they could enter clinical trials faster than new therapies, though gene therapies remain essential for a cure.