Did you know that childhood cancers are considered to be rare diseases?

As we approach Rare Disease Day at the end of February, we celebrate the achievements of the Zero Childhood Cancer Program (ZERO), which is now open to every Australian child with cancer, even those that we expect will respond well to conventional treatments, to help children like Scout.

Scout was born into a family of four. Her parents, Edwina and Sean, had two boys when they decided they’d like to have another baby. One day Sean came home from work and asked Edwina what was going on with Scout’s head. ‘It just looked different,’ he says. ‘It looked quite swollen.’

An MRI confirmed that Scout had a tumour. Edwina and Sean were advised that surgery was needed to relieve pressure on Scout’s brain. Following an 8-hour operation, Edwina and Sean were told everything had gone well, and the surgeon had removed as much tumour as he could see.

Pathology tests at the hospital revealed that Scout had a type of brain cancer known as a high-grade glioma, for which the standard treatment is chemotherapy, and Scout began chemotherapy.

Meanwhile, Scout was enrolled on the Zero Childhood Cancer Program (ZERO), a national precision medicine program, run in partnership by Children’s Cancer Institute (CCI) and the Kids Cancer Centre at Sydney Children’s Hospitals Network.

“ZERO is now open to every Australian child and young person with cancer, even those who we expect will respond well to conventional treatments,” says Associate Professor Mark Cowley, head of CCI’s Computational Biology Research Group. The Group was established with Luminesce Alliance support and is part of the ZERO team.

The Computational Biology Group, the largest of its kind in Australia, is able to process vast amounts of molecular data much faster, allowing informed treatment recommendations in real-time for children with cancer such as Scout.

The ZERO results confirmed that a targeted therapy called an ALK inhibitor could potentially be used to treat Scout’s tumour. This proved a godsend as scans revealed that seven months of standard chemotherapy had failed to stop Scout’s tumour from growing.

Scout began receiving the new drug in November 2023. In January 2024, scans showed her tumour had shrunk by 30%. Then a second scan in March revealed it was stable.

‘It was like a switch,’ Sean says. ‘From stopping chemo to starting the trial, it was like she was a completely different child. She was more active, more vocal, everything changed. And that was because of this treatment.’

Edwina says ‘For the first time, it feels like she’s a normal little baby,’ Sean agrees.

The computational biology team is currently undertaking the Advanced Genome and Transcriptome Analytics project as part of the Luminesce Alliance Data Enabling Platform, helping to further increase our capacity to help children like Scout. https://www.luminesce.org.au/research/current-research/advanced-genome-and-transcriptome-analytics/