An Australian research collaboration has identified several existing TGA‑approved medicines that could help slow brain damage in children with a rare form of dementia—offering hope for faster treatment options.
In a new study published in the prestigious Nature Communications journal, medical researchers used patient-derived brain cells grown in the lab, combined with advanced imaging and artificial intelligence, to rapidly test approved drugs and pinpoint those that improve brain cell health.
The research focused on Sanfilippo syndrome, a devastating childhood dementia that causes progressive loss of memory, behaviour and physical abilities. There are currently no widely available treatments.
Sanfilippo syndrome is one of more than 100 genetic disorders that together affect 1 in 2900 children in Australia and half of all children with dementia die by the age of ten.
To model the disease, researchers reprogrammed skin cells from affected children into brain cells that mimic how the condition develops. These cells reproduced key features of the disease, including toxic build-up, inflammation and ongoing cell loss.
Using this platform, the team screened 63 existing medicines and identified nine that significantly improved cell function within two weeks. Several of the drugs reduced brain cell damage, while others restored critical signalling linked to learning and behaviour. The findings also suggest that combining treatments may produce stronger, longer-lasting effects.
Lead researcher Flinders University’s Professor Cedric Bardy, who heads the Laboratory for Human Neurophysiology and Genetics at SAHMRI, and is the founding director of Brain Organoid Therapeutics, a cutting-edge preclinical screening platform derisking drug discovery, says the approach could dramatically accelerate treatment discovery.
“By combining human brain cell models with machine learning, we can quickly identify therapies that shift diseased cells toward a healthier state,” says Professor Bardy, whose research is leading the way in using human cells to better understand brain diseases.
“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.”
Because the drugs are already approved for other conditions, they could move into clinical trials much faster than entirely new therapies—bringing help to families sooner.
“Almost all childhood dementias have a clear genetic cause, and gene therapies will remain essential to cure the disease. However, identifying therapeutics that can manage daily symptoms and slow irreversible brain damage in children until a cure is discovered is urgently needed. ”
In another recent Nature Communications publication, the team demonstrated for the first time that brain cells from children with Sanfilippo become abnormally excited during early brain development and get stuck in overdrive.
In the newly published work, the team has now used their platform to identify a combination of repurposed drugs that can return brain cell activity to healthy levels.
“This is really important, because if translated clinically, it may help the children manage troubling symptoms such as hyperactivity, while also protecting them from irreversible neuronal loss,” says Professor Bardy.
Childhood Dementia Initiative CEO and founder Megan Maack was the driving force behind the project. Ms Maack and team secured the MRFF funding, assembled the researchers and managed the collaboration while CEO of the Sanfilippo Children’s Foundation (which has since merged with Childhood Dementia Initiative).
“This research is significant not just for Sanfilippo syndrome, but for the field of childhood dementia as a whole,” says Ms Maack. “It demonstrates that, with research, we can make progress for children with dementia, who have no targeted treatments or cures to date. What’s needed now is sustained and coordinated research funding across the pipeline to accelerate progress for all conditions that cause childhood dementia.”
Childhood Dementia Initiative continues to lead world-first action and awareness for all children with dementia, including those with Sanfilippo syndrome.
The research, initiated by Sanfilippo Children’s Foundation and led by Flinders University in collaboration with SAHMRI, the University of Adelaide and Women’s and Children’s Hospital, also provides a powerful new platform for testing treatments for other brain disorders, including Alzheimer’s disease.
While further studies are needed, the findings mark an important step toward faster, more practical treatments for childhood dementia.
Professor Cedric Bardy is an internationally recognised leader in the field of human neural models. His research efforts are focused on developing in vitro human brain tissue and new biotechnologies to unravel the effect of neurological disorders at the cellular, molecular and functional level.
The paper, ‘ Drug screen and machine learning predict neuroprotective agents in a preclinical human model of childhood dementia’ , by Zarina Greenberg, Ella McDonald, Alejandra Noreña Puerta, Manam Inushi De Silva, Cade Christensen, Robert Adams, Jenne Tran, Paris Mazzachi, Sebastian Loskarn, Siti N Mubarokah, Leanne Winner, Drew Neavin, Megan Maack, Kristina L Elvidge, Lisa Melton, Mark R Hutchinson, Kim M Hemsley, Nicholas Smith and Cedric Bardy, was published in Nature Communications. DOI: 10.1038/s41467-026-76837-1.
Collaborating organisations included Flinders University, SAHMRI, Adelaide University, the Women’s and Children’s Health Network, the Childhood Dementia Initiative, Sanfilippo Children’s Foundation and Cure Sanfilippo Foundation.
Read more here: https://news.flinders.edu.au/blog/2026/04/08/childhood-dementia-cause-in-the-crosshairs/
Acknowledgements : This work was supported by the Australian Medical Research Future Fund (MRFF) Accelerated Research Program (EPCD000025) granted to the Sanfilippo Children's Foundation (LM, KE, MM, KH, NS, MRH, CB) with additional funding from Sanfilippo Children’s Foundation, The Australian Medical Research Future Fund (MRFF) Stem Cell Therapies Mission (CB, MRF2024419) and The Grosset Gaya Fund (CB).
Nature Communications
Computational simulation/modeling
Cells
Drug screen and machine learning predict neuroprotective agents in a preclinical human model of childhood dementia
20-Aug-2026