Dialog Box


Australian kids among first to access new clinical trial

Kayne and Danii Cook share their brain cancer story. 

Australian children with aggressive brain cancer will be among the first to benefit from an international clinical trial for nivolumab; a new immunotherapy drug, which has shown promise in other aggressive cancers.

In an Australian first, experts from the Children’s Cancer Institute and Sydney Children’s Hospital, Randwick will partner with The Hospital for Sick Children in Toronto, Canada, to trial the drug’s effectiveness on children with hypermutated tumours; which are tumours with an extremely high number of tumour mutations.

The trial will open next year in Sydney, Brisbane and Adelaide, in partnership with the Australian and New Zealand Children’s Haematology and Oncology Group (ANZCHOG).  

Nivolumab works by stimulating the body’s own immune system to fight cancer, targeting and blocking a protein called PD-1 on the surface of certain immune cells called T-cells. Blocking PD-1 activates the T-cells to find and kill cancer cells. 

The Toronto group has already successfully treated two siblings with a congenital biallelic mismatch repair deficiency (bMMRD) tumour, which is usually fatal.

This international trial will provide the first data to examine the use of this checkpoint inhibitor in children with recurrent and refractory hypermutated brain cancer; potentially providing a new treatment option for these patients.

Eligible patients will be identified through the Zero Childhood Cancer Program genetic testing platform, a personalised medicine program led by Children’s Cancer Institute and Sydney Children’s Hospital.

Part of a $5 million commitment to brain cancer research by the Medical Research Future Fund’s Brain Cancer Mission, together with $86,000 from Cure Brain Cancer Foundation, will be used to enable 15 children to join the pilot trial. 

This takes Cure Brain Cancer Foundation's investment in paediatric brain cancer-related projects over the last five years to more than $7.7 million.

Associate Professor David Ziegler.

According to Associate Professor Ziegler, paediatric oncologist and study lead at Sydney Children’s Hospital, the trial is about matching the right patient to the right treatment to achieve the best possible result. 

“Immunotherapy is a powerful new treatment for many types of cancer, but not everyone responds,” said A/Prof Ziegler.

“We have treated several patients with ‘hypermutated’ tumours and seen dramatic effects. We are now partnering with our colleagues in Canada to expand access to this breakthrough treatment to children from around Australia based on an in-depth genetic profiling of their tumour.”

Michelle Stewart, CEO of Cure Brain Cancer Foundation, praised this international project. 

“We are proud to fund this international collaboration, and give some of Australia’s youngest and most vulnerable brain cancer patients access to a promising new trial. International collaboration greatly increases understanding of rare and deadly types of brain cancer, and it is through collaborations like this we will find better treatment options for all Australians diagnosed with this brutal disease in the future.” 

The Zero Childhood Cancer Program has received close to $7 million from the NSW Government - part of its $22 million investment into paediatric cancer research over the last five years. 

Help fund vital brain cancer research