Despite the great improvement in survival rates for childhood cancer over recent decades, a number of malignancies remain refractory to standard chemotherapy. Amongst these is neuroblastoma, the commonest solid tumour of young children, for which alternate therapies are urgently required. We have identified and validated a number of key genes associated with the malignant phenotype of neuroblastoma, and then either identified existing small molecule drugs which can be used in new ways to treat this disease, or are developing new small molecule drugs using high-throughput screening of chemical small molecule libraries. This research has led to a number of current and planned clinical trials for children with this disease, as well as the development of lead compounds in partnership with commercial partners and licensing of specific compounds for use as tools in research.
In addition, we have established the Zero Childhood Cancer national child cancer personalized medicine program, in partnership with the Kids Cancer Centre at Sydney Children’s Hospital, which is using whole genome sequencing of tumour and germline samples, RNA sequencing, methylome analysis and deep sequencing based on targeted panels, together with in vitro and in vivo drug response testing, to identify personalized treatments for children throughout the country with less than 30% chance of survival. This national clinical trial involves all eight Australian paediatric oncology treatment centres, and 13 national and international research partners, and is delivering tailored treatments for high risk patients in real time.