Skip to content
The Kids Research Institute Australia logo
Donate

Discover . Prevent . Cure .

Finding new treatments for rare brain cancers in infants

The WA Kids Cancer Centre has secured $1.1 million in funding from the Medical Research Future Fund’s (MRFF) Paediatric Brain Cancer Research Stream 2 to develop more effective and less toxic treatments for rare brain cancers in infants.

nick gottardo and raelene endersby

Pictured: Professor Nick Gottardo and Associate Professor Raelene Endersby

The WA Kids Cancer Centre has secured $1.1 million in funding from the Medical Research Future Fund’s (MRFF) Paediatric Brain Cancer Research Stream 2 to develop more effective and less toxic treatments for rare brain cancers in infants. 

Rare brain cancers in babies and toddlers are almost always fatal. Many of these cancers mostly affect children under three years old, whose developing bodies cannot tolerate the intense treatments used for older children or adults. The outcomes of treatment are often devastating, highlighting the urgent need for improved therapeutic options. 

Associate Professor Raelene Endersby, Co-lead of the WA Kids Cancer Centre Brain Tumour Research Team and lead Chief Investigator on the project, emphasised the lack of evidence-based treatment options for these vulnerable patients. 

“Alarmingly, there are no active clinical trials in Australia focused on these cancers due to their rarity. Our research aims to change this by building a strong evidence base to support new clinical trials for infants and very young children,” Associate Professor Endersby said. 

To cater for the unique challenges of treating infants, whose developing systems respond differently to treatment than older children or adults, Associate Professor Endersby’s team has developed advanced, age-appropriate childhood cancer models. 

“We have created models that mimic the growing brains of infants and demonstrate critical differences in how cancers respond to treatments in younger bodies. 

“These models allow us to better understand the efficacy and safety of potential new therapies for infants and young children.” 

Using these world-first childhood cancer models, Associate Professor Endersby’s team will test new treatments and identify those that have the best chance of success in clinical settings.

“Upon completion of the project, we will have optimised treatment regimens for multiple rare brain cancers, proven their safety, and established an evidence base for future clinical trials.  

“We hope to see our findings translate into the clinic and provide urgently needed options for infants diagnosed with high-risk brain cancers.” 

Landmark $14 million national partnership for paediatric brain cancer trials

In addition to the Stream 2 project, the WA Kids Cancer Centre will also contribute to an initiative under the MRFF’s Paediatric Brain Cancer Research Stream 1, which has received $14 million in funding over seven years.

This grant, led by from Co-head of the WA Kids Cancer Centre Brain Tumour Research Team Professor Nick Gottardo, with Associate Professor Raelene Endersby, aims to integrate preclinical and clinical brain cancer research across Australia.

This will be executed through the establishment of the Consortium for Australian Children’s Trials in Brain Cancer (CoACT: Brain Cancer). CoACT unites 77 investigators from over 30 institutions, including members of the Australian and New Zealand Children’s Haematology/Oncology Group (ANZCHOG), leading cancer research groups, international clinical trial consortiums, and all nine Australian paediatric oncology departments.

The consortium focuses on clinical trials, survivorship, quality of life, neuropsychology, radiation oncology, bioinformatics, and consumer advocacy. Its mission is to increase clinical trial opportunities for children with brain cancer and improve patient outcomes.

This initiative provides the platform to expedite translation of research discoveries, including those from A/Prof Endersby's laboratory-based project, into clinical applications that could transform outcomes for children diagnosed with rare brain cancer.