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 unstable DNA. A subset of genes called long non-coding RNAs (lncRNAs) are the most diverse class of genetic molecules in cells. Once believed to be “junk DNA”, recent research has shown that they play a role in cancer development and growth and that their expression is highly specific to cancer cells. 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.