Researchers at Queensland Institute of Medical Research (QIMR Berghofer) are looking for drugs that can slow or halt the progression of childhood dementia. The research will focus on a range of childhoood dementia disorders.

“This is a great development and is the kind of research we’re advocating for. Historically, research was applied to just one single childhood dementia disorder. By focusing on a number of disorders concurrently, this project has the potential to benefit more children. It’s also more efficient because the infrastructure and resources for this project will be established once for multiple disorders, rather than recreated and replicated to study just one single disorder at a time,” says Dr Kristina Elvidge, Head of Research at Childhood Dementia Initiative. 

Researchers at QIMR Berghofer started their research on one type of childhood dementia, Batten disease, with a grant from the Batten Disease Support and Research Association Australia (BDRSA). Now, thanks to a philanthropic donation, researchers are extending their drug discovery pipeline to a range of other childhood dementia disorders including Tay-Sachs disease, Hurler syndrome, Niemann-Pick C and Sanfilippo syndrome. They plan to add more types of childhood dementia in the future.

The project will use computer-based algorithms to analyse the genetics of childhood dementia and identify promising drugs for repurposing as potential treatments. These drugs will then be tested on tiny ‘model brains’ (brain cells from children with dementia cultivated in a lab) to assess the drugs’ impact on childhood dementia. 

This project also brings together a great collaborative team. Associate Professor Anthony White who is leading the project has 25 years of experience in neurodegenerative disease research and drug development including for Alzheimer’s and motor neurone disease. Professor Eske Derks and Dr Zac Gerring at QIMR will contribute their expertise in computational biology and drug repurposing. They will also collaborate with Professor Tony Cook at University of Tasmania, Wicking Dementia Centre who has expertise in generating cell models.

“As the project focuses on repurposing drugs already in use, drug approval time frames can be reduced from 10 years to 1 or 2 years. This is because the drugs don’t have to go through the same lengthy and costly clinical trial process that brand new drugs do,” said Anthony White. “We hope to find drugs that could be used to treat a range of childhood dementia to bring benefits quickly and cost-effectively to larger numbers of affected children.” 

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