Brain cancer

Brain cancer causes the most childhood cancer deaths due to its location in the body and lack of treatment options.

I believe we can do for brain cancer what has already been done for leukaemia. Once the survival rate for acute lymphoblastic leukaemia was 0%, today it’s 90%.


Most chemotherapy drugs don’t cross the blood-brain barrier, and so can’t reach tumour cells. Surgery may be impossible due to the sensitive location of the cancer.

The brain and the spinal cord make up the central nervous system. Several different types of cancer can arise here and are named after the part of the brain or spinal cord in which they are located, or the type of cell from which they develop.

Main types in childhood

Two common types of brain cancer in children are medulloblastoma and glioma. Medulloblastoma arises in the cerebellum, at the back of the brain, an area that controls movement and coordination. Glioma develops from glial cells.

The most common high-grade glioma in childhood is Diffuse Intrinsic Pontine Glioma (DIPG). DIPG is the most aggressive and devastating of all childhood malignancies. Almost all children diagnosed have a short history of symptoms, experience rapid tumour progression and do not survive beyond 1–2 years of diagnosis. Surgery is not possible due to the tumour’s sensitive location within the brainstem. Patients do not respond to chemotherapy and radiation therapy only briefly delays tumour progression. Because of this, new approaches are desperately needed and our research in this area is vitally important.

For more information about brain cancers, see the websites of:

Please note that, while Children’s Cancer Institute conducts medical research into childhood cancer and works closely with clinicians, we do NOT treat patients and unable to provide treatment advice. For questions or concerns about an individual child’s cancer treatment, or to find out about relevant clinical trials, please contact your treating oncologist (cancer specialist).

Amy’s story

When Ren and Sharon’s daughter Amy was diagnosed with terminal cancer at just 7 years old, she went from a bounding ‘Duracell bunny’ to losing her ability to walk and speak in under 18 months. Read Amy’s story.

Our brain cancer research

Examples of our brain cancer research teams, achievements and projects include:

  • A/Professor David Ziegler leads our Targeted Therapy group, the only laboratory program in Australia dedicated to researching and finding new treatments for Diffuse Intrinsic Pontine Glioma (DIPG). Children’s Cancer Institute is the Australian hub of the international DIPG registry, working with DIPG researchers from around the world.
  • A critical problem for research in improving treatments has been lack of surgical tissue from biopsies. To solve this, we initiated a national DIPG tumour donation protocol. With tissue donated from autopsies, we have grown DIPG cells in the laboratory as they grow in the brain (in ‘neurospheres’) to investigate DIPG’s biology and test new therapies. In the past three years, we have screened over 3,700 pharmaceutical compounds and identified a number of exciting potential new treatments. We’re looking to change the future of children with brain cancer.
  • Researchers in our Tumour Biology and Targeting program led by Professor Maria Kavallaris are investigating the potential of nanotechnology to create better therapies for medulloblastoma, designing polymers to package and deliver therapeutic drugs or genetic material to tumour cells. We are also researching the highly malignant and invasive brain tumour glioblastoma to find new therapeutic targets for future treatments.
  • The Zero Childhood Cancer national personalised medicine program, led by Children’s Cancer Institute in partnership with Sydney Children’s Hospitals Network, opened a national clinical trial in late 2017. It includes children with the most serious cases of infant, childhood and adolescent cancer. Children with brain tumours made up around half of all the patients in our pilot study.