Targeting childhood cancer where it begins

Background

Childhood cancer has enormous impact on children, families and communities, and is the most common cause of childhood death from disease. In Australia, almost 200 of the 1500 children and adolescents diagnosed with cancer each year will be classified as high-risk after initial treatment with chemotherapy and, sadly, most will die of their disease. More effective therapeutic strategies are urgently needed for the treatment of children with these aggressive cancers. 

The majority of childhood cancers are believed to be embryonal, which means the cancer begins in stem cells that are left behind from when a child first develops in the womb. A protein, called MYCN, has been shown to play an important role in driving the development of neuroblastoma (an aggressive childhood cancer) by stopping the normal differentiation of the embryonal stem cells. Professor Murray Norris’s team has discovered that blocking a particular gene, called RUNX1T1, can interrupt MYCN activity and prevent the development of neuroblastoma. 

The Research

In this project, Professor Murray Norris and his team will study RUNX1T1, building a detailed understanding of its role in maintaining normal function of embryonic stem cells. With this knowledge, the team will look at targeting the gene, to find out if stopping RUNX1T1 can make cancer more visible to the immune system. In preclinical tests, they’ll investigate combining RUNX1T1 knockdown with existing chemotherapy and immunotherapy treatments to see if they can enhance their cancer-fighting effectiveness.