Developing less toxic treatment approaches for childhood brain cancer

Brain Cancer

Lead Researcher: Dr Nicholas Gottardo

Institution: Telethon Kids Institute

Cancer Council NSW Funding: $197,432

Funding Duration: 2020 – 2021


Brain cancer kills more children than any other disease in Australia, affecting up to 200 children every year. The most common childhood brain cancer is called medulloblastoma and up to 60% of children with high-risk disease will die. Those who do survive often face lifelong side-effects including developmental issues, heart problems, hearing loss and increased risk of developing other cancers later in life due to highly toxic chemo- and radio-therapies used to treat their disease. Current treatment has not changed in 30 years. New and effective therapies are urgently needed to improve the survival and quality of life of children with medulloblastoma. 

The Research

Dr Nicholas Gottardo is developing a less toxic therapy which uses the patient’s own immune system to fight the brain cancer cells. The aim is to improve survival and minimise the long-lasting side effects associated with medulloblastoma treatment. 

The team has already shown this immunotherapy significantly extended survival in medulloblastoma pre-clinical models with minimal side effects. In this project, Dr Gottardo and his team will further test the immunotherapy in pre-clinical models to prove this new treatment will work in combination with current medulloblastoma treatments. Using two different models of high-risk medulloblastoma, the team will mimic current clinical treatment protocols – using radiotherapy – and combine their immunotherapy with standard treatment. 

The Impact

Dr Gottardo and his team hope their new and exciting therapy will provide the answer to minimising devastating side effects associated with medulloblastoma treatment. This project is a crucial step for the future development of a clinical trial of this new treatment approach. By proving that their new immunotherapy can be safely combined with current treatments, they will be able offer patients the opportunity to participate in a clinical trial earlier – rather than waiting for the standard treatments to fail.

This will increase patient survival rates, as well as improving the quality of life of children affected by brain cancer. If successful, the team want to extend their findings to other childhood brain cancers with poor outcomes.


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