Improving outcomes for a devasting childhood leukaemia
Background
Each year, around 50 Australian children are diagnosed with Acute Myeloid Leukaemia (AML) – a particularly devasting blood cancer for children. While there have been significant improvements in survival across most childhood blood cancers, the 5-year survival rate for children with AML has plateaued at around 70%. The standard treatments for childhood AML are high-intensity chemotherapy and stem cell transplantation. These potent treatments are also toxic to healthy cells and cause side effects that impact a child’s quality of life. More effective and less damaging treatments are needed to improve outcomes for children with AML.
The research
Cancer is a complex disease characterised by abnormal gene behaviours and an unstable genome. Long non-coding RNAs (lncRNAs) are genetic molecules that are often expressed specifically in cancer, and recent research has shown that they can even drive the development of cancer across many different cancer types. This makes lncRNAs an ideal target for potential new cancer treatments that will spare healthy cells of the body. Professor Lock leads one of the few teams globally who are investigating the treatments for targeting lncRNAs in paediatric AML.
In this project, Professor Lock and his team will investigate lncRNAs specifically in childhood AML. Using patient information, robust genetic data, pre-clinical models and gene-silencing technologies, the team will identify the non-coding RNAs in childhood AML and test different approaches for switching off their capacity to drive cancer growth.
The Impact
The study of lncRNAs is still an emerging area of cancer research. The findings generated from this study will significantly influence this field of research and provide new treatment approaches for childhood AML. If the team’s research is successful, they will be well-placed to progress to a clinical trial for a much–needed and less toxic treatment with the potential to improve survival and quality of life outcomes for children with this devastating cancer.
Project update 2026
This project has identified a promising new target for treating childhood AML. The team was the first to show that the gene SLX1B-SULT1A4 is critical for leukaemia cell survival. Importantly, this gene is found at much higher levels in cancer cells than in healthy blood cells, making it a promising target for more precise treatment. By identifying a potential way to distinguish cancer cells from healthy cells, this research opens the door to therapies that could improve survival while reducing the serious side effects associated with current chemotherapy.