Existing Drugs Could Treat Childhood Dementia, Australian Study Finds

Genetic mutations can cause children to lose memory and mental processing much as older adults do in age-related dementia. Australian researchers say drugs already approved for other diseases could help treat these childhood dementias, according to a study published in the journal Nature. Because childhood dementias are usually caused by a mutation in a single gene, researchers can skip straight to testing whether existing, already-safety-tested drugs work against them, rather than developing new treatments from scratch.

The researchers, led by senior author Cedric Bardy, a neurologist at the South Australian Health and Medical Research Institute, focused on Sanfilippo syndrome, the most common form of childhood dementia. It is caused by a defective gene for an enzyme that breaks down a carbohydrate called heparan sulfate, leading to a toxic buildup in tissues including brain cells; few children with the condition survive beyond their teenage years.

Bardy's team grew neurons from skin cells donated by children with Sanfilippo syndrome, recreating the disease's heparan sulfate buildup, inflammation and cell death in the lab. Using machine learning, imaging and other techniques, they screened dozens of existing drugs and identified at least nine promising compounds; some reduced the toxic buildup, others extended cell lifespan or improved communication between cells.

Some of the identified drugs are already approved for other uses, including the gout medication probenecid and the pain reliever ketorolac. "Almost all childhood dementias have a clear genetic cause, and gene therapies will remain essential to cure the disease," Bardy said, adding that therapies to manage symptoms and slow brain damage until a cure is found are urgently needed.