Repurposed Drugs Show Promise for Childhood Dementia
Researchers have found options for childhood dementia using existing medicines. The study focused on Sanfilippo syndrome, a genetic disorder that causes brain damage in children.
Scientists used brain cells created from patients’ skin cells. These lab-grown cells copied disease features, including toxic buildup, inflammation, and cell loss.
The team tested 63 medicines approved by Australia’s Therapeutic Goods Administration. Nine improved brain cell function within two weeks. Some reduced cell damage, while others restored signals linked to learning and behaviour.
AI Speeds Up Drug Testing
Researchers combined imaging with artificial intelligence to study cell changes. This method helped identify medicines that moved unhealthy cells toward healthier activity.
The study also found that treatment
combinations may provide benefits. In addition, Sanfilippo brain cells can become unusually active during early development.
Researchers identified a combination that helped return brain activity to healthier levels. This finding could manage symptoms such as hyperactivity while protecting nerve cells.
Hope for Faster Treatments
Because these medicines have approval for other conditions, researchers may advance them faster. However, testing must happen before doctors can use them for childhood dementia.
Professor Cedric Bardy from Flinders University led the research with institutions. The collaboration included SAHMRI, the University of Adelaide, and Women’s and Children’s Hospital.
Researchers say gene therapy remains important because genetic disorders need solutions. Still, existing medicines could help slow brain damage while scientists work toward cures.
Wider Impact on Dementia Research
The research could support studies for other brain disorders. Scientists believe the platform may help test therapies for conditions such as Alzheimer’s disease.
For families affected by Sanfilippo syndrome, the findings offer hope. Yet the results came from laboratory models, not clinical trials.
Therefore, researchers must confirm safety, dosage, and effectiveness in people. The study marks an important step toward faster treatments for children with dementia.
