International Childhood Cancer Day
February 15 is International Childhood Cancer Day, a day when we pause to consider the impact of childhood cancer on children and families around the world, and the global battle to improve outcomes for these patients.
The past few decades have seen tremendous success in our many battles against this disease. One of the greatest success stories in modern medicine has been the improvement in cure rates for children with acute lymphoblastic leukaemia, the most common childhood cancer. Once considered incurable just 50 years ago, survival rates now exceed 90%, thanks to intensive chemotherapy and, more recently, powerful immunotherapies that further improve outcomes.
Yet alongside this success, there are areas where progress has been far more limited. After leukaemia, brain cancer is the next most common childhood cancer and remains the leading cause of disease-related death in children worldwide. There are some brain cancers that have no effective treatments from the moment they are diagnosed. The worst of these is known as diffuse midline glioma, or DMG. Other common brain cancers, such as medulloblastoma, are potentially curable but at a heavy cost, with many long-term side effects. If they recur, there are often no effective options.
For many years, progress in brain cancer was constrained by a lack of tools, funding and access to tumour samples. Researchers often did not even have tumour samples available, nor the funding, nor the cutting-edge techniques needed to understand these diseases properly.
Times have changed. Parent-led organisations around the world have raised millions of dollars to close funding gaps. Tumour samples are now available through donations and through biopsies made possible by advances in neurosurgical techniques. Rapid advances in genomic technologies mean that scientists have powerful new tools at their disposal.
Taken together, this means we are entering an era of renewed optimism and hope. Indeed, in some low-grade brain tumours, the idea of controlling or even eliminating disease with a tablet taken once a day or once a week is becoming reality. New targeted therapies are already approved or in development for children whose tumours previously had few treatment options. For the more aggressive, high-grade tumours, we face greater challenges, but we also have more hope than ever before. As we have seen with leukaemia, new immunotherapies are, for the first time, producing meaningful responses in some tumours where previously no options were available.
There is growing confidence that through collaboration between scientists, clinicians and families, we can fundamentally change the outlook for children with brain cancer. To achieve this, sustained and increased funding from governments and the broader community is essential to ensure we can continue to build on and accelerate these discoveries.
Professor David Ziegler is a paediatric oncologist at the Kids Cancer Centre, Sydney Children’s Hospital, Randwick. He trained as a Fulbright Scholar at Harvard Medical School. He is Head of the Neuro-Oncology program and the Cancer Clinical Trials program at SCH. He is Group Leader of the Brain Tumour Group at the Children’s Cancer Institute, Australia, and Conjoint Professor at the University of New South Wales. He leads multiple national and international clinical trials for children’s cancer, including Australia’s childhood cancer personalised medicine trials through the ZERO program. He has won multiple awards, is an NHMRC Investigator, and a Fellow of the Australian Academy of Health and Medical Sciences.


