Crucial step in hunt for childhood dementia treatment

A team of Adelaide researchers, recognised globally for a unique drug-screening project aimed to improve symptoms for those with the disease, has been awarded a two-year, $730,000 grant from the Australian government’s Medical Research Future Fund (MRFF), to further their work.

Dubbed the Brain in a Dish project, SAHMRI and Flinders University Associate Professor Cedric Bardy leads the team that has been working since 2019 to grow brain cells in the lab from children with Sanfilippo syndrome.

Researchers are comparing the appearance and function of the Sanfilippo cells to cells from healthy children, and using them to screen a panel of already available drugs to identify potentially effective therapies.

“We hope to repurpose drugs initially designed for more common adult dementia and neurodegenerative disorders,” Prof Bardy said.

“If our strategy works, we will rapidly translate this breakthrough into a clinical trial and provide some relief to the families affected … we are working extremely hard to make a breakthrough and help improve the quality of life for the children and families living with this horrific disease."

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