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The recipients of $2.7 million in research funding for childhood dementia were announced by Australia's Federal Minister for Health and Aged Care, Mark Butler. The announcement was made on the morning of the world's second Childhood Dementia Symposium, held in Sydney on 14 March 2023. This is the first funding specifically directed to childhood dementia in the world, and will pave the way for improvements in the treatment of childhood dementia. 

Funding was awarded to five cutting-edge research projects aiming to develop new treatments for childhood dementia:

  • Associate Professor Anthony Cook from the Wicking Dementia Research & Education Centre at the University of Tasmania will develop and test a new substrate reduction strategy to treat childhood dementia. In collaboration with researchers at Murdoch University in Perth they will investigate if this treatment approach can stop the build up of certain harmful substances in the brain. It will be tested in cell models of around 10 different types of childhood dementia.
  • A multidisciplinary team at Sydney Children’s Hospital Network, John Hunter Children’s Hospital, UNSW Sydney and University of Sydney, led by Professor Michelle Farrar will develop a comprehensive biomarker panel for childhood dementia. Biomarkers are substances in bodily fluids such as blood or spinal fluid, that can be used to understand disease processes, monitor disease progression and measure the benefit of treatments
  • Dr Ya Hui Hung at The Florey in Melbourne will develop an mRNA-based gene therapy for Niemann-Pick Disease Type C1. This project, being conducted with collaborators at Royal Melbourne Hospital and Monash University, will serve as a blueprint to establish an mRNA-based gene therapy pipeline for other types of childhood dementia.
  • Associate Professor Wendy Gold’s project aims to uncover new therapeutic targets, disease drivers and biomarkers for Rett syndrome. The researchers at the University of Sydney and the Children's Medical Research Institute will use patient cell models to increase understanding of this complex disease and accelerate improved treatment options for individuals with Rett syndrome.
  • Dr Nicholas Smith from University of Adelaide and the Women’s and Children’s Hospital, with collaborators at Flinders University and University of South Australia will develop nanoparticle technology as a platform for targeted gene-delivery for Sanfilippo syndrome. This technology has wide applicability across the childhood dementia conditions if proven successful.

The announcement also included another project for childhood dementia funded out of the Medical Research Future Fund’s Stem Cell Therapies Mission. This is another $730k for a project at Flinders University to repurpose drugs for Sanfilippo syndrome.

“This is a great step not just because more funding for childhood dementia research is urgently needed, but because these projects demonstrate the growing adoption of new approaches to childhood dementia research. Instead of the traditional approach of studying one individual type of childhood dementia in isolation, these projects either focus on multiple childhood dementia conditions at once, or will have outcomes that can benefit childhood dementia broadly. Research of this kind will accelerate progress for children,” said Dr Kris Elvidge, Head of Research, Childhood Dementia Initiative.

Childhood Dementia Initiative worked with researchers who submitted applications for funding. The Initiative ensured that family and patient voices were heard and considered in proposals, connected researchers and experts who could collaborate, and encouraged research projects that focus on priority areas.

“We were delighted to discuss this progress at our Childhood Dementia Symposium which brought together 95 scientists, clinicians, industry representatives and family advocates this week. We look forward to working with the researchers on these important projects,” said Dr Elvidge.

“This is a much welcomed boost to childhood dementia research, but this is just the start. Childhood dementia is still woefully underfunded compared to other conditions such as childhood cancer which causes a similar number of deaths each year. We will continue to advocate for more funding that will lead to the scientific breakthroughs needed to give children with dementia brighter futures.” said Megan Maack, CEO, Childhood Dementia Initiative.

This Medical Research Future Fund grant opportunity was first announced at the inaugural Childhood Dementia Symposium in 2022. To hear more about these projects, other new childhood dementia research advances, and to take advantage of networking opportunities, please sign up to the Childhood Dementia Research Alliance.