Next Generation Precision Medicine Program

Overview

Program Lead | Professor Ron Firestein

Addressing unmet needs in childhood cancer treatment

Genomic sequencing has the potential to reveal mutations that can predict a child’s response to targeted cancer therapies. However, actionable mutations are identified in less than 20% of children with cancer, and of these, only half respond to available therapies. This leaves a significant gap in effective treatment options, particularly for childhood cancers with the lowest survival rates, such as soft tissue tumours and brain cancers. There is an urgent need for new, precise treatments to improve outcomes for these young patients with high-risk malignancies.

Advancing childhood cancer treatment

The Next Generation Precision Medicine Program is identifying the next wave of paediatric cancer-targeted therapies through a comprehensive, child-centric approach that includes

1. Generation of novel models: Creating new models of childhood cancers that faithfully represent the patient’s tumour.
2. Multi-omics characterisation: Analysing these models at multiple levels (genome, transcriptome, epigenome, proteome) to uncover unique tumour features.
3. Functional genomic screens: Conducting extensive functional genomic screens to identify the genetic drivers and dependencies of low-survival paediatric cancers.
4. Childhood Cancer Model Atlas: Developing a data portal that enables integrative genomic analyses at a cohort level, providing valuable insights for researchers and clinicians.