Australian researchers screen drugs for Sanfilippo syndrome using skin cells
Australian researchers have found new hope for children with rare genetic brain disorders like Sanfilippo syndrome, a type of childhood dementia that causes toxic buildup in the brain and leads to early memory loss.
By turning skin cells from affected children into lab-grown neurons, they recreated the disease in the lab and used smart tech to test which existing medicines might help.
Researchers identify 9 promising drugs
Out of all the drugs tested, at least nine showed real promise: some cleared out harmful buildup, while others helped brain cells live longer or talk to each other better.
Two Food and Drug Administration-approved medicines, probenecid and ketorolac, have existing FDA approval for other conditions.
As senior author Cedric Bardy put it, "Our findings give us confidence that we can make a meaningful difference in the lives of children affected by these devastating disorders and turn those discoveries into treatments that can improve lives."
This approach could even help tackle other forms of childhood dementia that arise from single-gene mutations down the line.