Leukaemia is the most common cancer in children. It represents about one third of all childhood malignancies.

Breakthroughs like PR-104 convince me that our work will find better treatments for the different types of leukaemia. It’s vital that we do.

Blood cells from leukaemia patient

Unlike other cancers, leukaemia does not form a solid tumour. It is a cancer of the blood and bone marrow, where large numbers of abnormal white blood cells accumulate and circulate in the body. This affects production of the other blood cells – red blood cells (that carry oxygen), platelets (that help clot blood), and normal white blood cells (that fight infection).

Main types in childhood

The main types of childhood leukaemia are acute lymphoblastic leukaemia (ALL) and acute myeloid leukaemia (AML). ALL mostly occurs in young children (1–10 years old) and is the most common type of childhood leukaemia, with around 150 Australian children diagnosed each year.

Within these two main leukaemia types there are at least 20 subtypes, with new ones continually being identified as a result of research. Sixty years ago, cancer was a death sentence for a child but now survival rates, especially for childhood leukaemia, are on average over 80%. But some leukaemia subtypes with specific genetic abnormalities or mutations have much lower survival rates, are less responsive to therapy and are far more likely to relapse. They include early T-cell precursor ALL (ETP-ALL), infant mixed lineage leukaemia-rearranged ALL (MLLr-ALL) and Ph-like (Philadelphia) ALL.

For many children with these high-risk leukaemia subtypes and with relapsed ALL, there are very few effective treatment options. This is why our research is vital to identify and develop more effective, targeted therapies.

For more information about leukaemia, 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 are 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).

Ruby’s story

Before her first birthday Ruby endured six gruelling rounds of chemotherapy, major surgery and countless lumbar punctures. Ruby has acute myeloid leukaemia (AML), and her future is uncertain. Read Ruby’s story.

Our childhood leukaemia research

Examples of our leukaemia research groups, achievements and projects include:

  • Professor Richard Lock leads our Leukaemia Biology Program, which investigates how childhood leukaemia develops resistance to conventional chemotherapeutic drugs, rapidly tests new drugs to prioritise for clinical trials, and identifies new targets to develop a new generation of more effective and specific leukaemia drugs which could include the drug PR-104.
  • For over a decade, Children’s Cancer Institute has been the leukaemia testing site for the Pediatric Preclinical Testing Consortium, supported by the US National Cancer Institute. We are the only non-US testing site in the Consortium, aimed at prioritising effective drugs for clinical evaluation in paediatric cancers.
  • While clinical remission is achieved initially in almost all acute lymphoblastic leukaemia (ALL) patients, about one fifth later suffer a relapse due to the persistence of small numbers of cancer cells throughout treatment (‘minimal residual disease’ or MRD). We have developed one of the best methods to measure MRD for ALL patients. Our Minimal Residual Disease group led by A/Professor Rosemary Sutton researches improvements in MRD testing methods and provides MRD diagnostic testing for ALL patients in hospitals around the country and enrolled on several clinical trials. Thanks to MRD and a 10-year trial called study 9, survival rates for high risk ALL have doubled from 35% barely 10 years ago to 70%, and more than 40 children are alive today who would have otherwise succumbed to their disease.
  • Preliminary studies by our Cancer and Stem Cell Biology group led by Dr Jenny Wang have identified several new modifiers of chromatin, the complex of DNA and proteins that forms chromosomes within the nucleus. These contribute to leukaemia formation from stem cells and to disease progression. Further research will identify targets crucial for leukaemia stem cell survival with potential to develop new cancer therapies.
  • Children whose leukaemias display abnormalities of the mixed lineage leukaemia (MLL) gene have particularly poor outcomes. In our Molecular Diagnostics program, we have established a pipeline for the discovery, characterisation and development of new MLL drugs, including molecular characterisation, in vivo testing in patient-derived xenograft (PDX) models, and their combination with leukaemia drugs currently in use.
  • The Zero Childhood Cancer national personalised medicine program, led by Children’s Cancer Institute in partnership with and Sydney Children’s Hospitals Network, opened a national clinical trial in late 2017. The pilot study for this clinical trial enrolled nearly 60 children, far in access of the 12 originally planned, due to strong demand from parents and doctors. The program includes children and adolescents with high-risk or relapsed cancer, including leukaemia.