Next generation precision medicine
Current precision medicine programs focus on genomic sequencing to identify discrete mutations that may predict patients’ responses to targeted therapies. Unfortunately, fewer than 20% of cancer patients harbour actionable mutations, and of those who do, only 50% respond to therapy. This underscores a clear need to go beyond genomic sequencing to identify new targeted therapies for paediatric cancers of poorest survival (i.e brain and soft tissue tumours).
The VPCC Next generation precision medicine program aims to identify the next generation of paediatric cancer-targeted therapies through a multi-pronged, paediatric-centric approach that encompasses:
- Generation of novel models of childhood cancers that faithfully represent the patient’s tumour;
- Characterisation of models at a multi-omics level (genome, transcriptome, epigenome, proteome);
- Comprehensive functional genomic screens to identify the genetic drivers and dependencies of low-survival paediatric cancers; and
- Development of a childhood cell line atlas and data portal to enable cohort-level integrative genomic analyses.
Importantly, paediatric models in VPCC research are coupled to patients whose clinical and molecular data is tracked (in partnership with the Zero Childhood Cancer program). This offers a unique opportunity to correlate patient responses in the clinic/clinical trial setting with both molecular variants and functional dependencies identified in the patient’s avatar model.
- Prof Ron Firestein – Hudson Institute of Medical Research
- Prof David Eisenstat – The Royal Children’s Hospital/Murdoch Children’s Research Institute
- A/Prof Peter Downie AM – Monash Children’s Hospital
- A/Prof Sefi Rosenbluh – Monash University
- Dr Jason Cain – Hudson Institute of Medical Research
- A/Prof Misty Jenkins AO – WEHI (Walter and Eliza Hall Institute)
- A/Prof Paul Ekert – Children’s Cancer Institute
- Prof Roger Daly – Monash University
On a broader level, the rarity of childhood cancers, paucity of suitable tumour material and lack of reliable preclinical models particularly highlight the need to develop a repository of well characterised and faithful models of childhood cancer. As such, the VPCC childhood cancer atlas will include a searchable data portal to enable the dissemination of patient models and computational analysis of multi-omic data sets. Together, these aims will enable identification and progression of the next-generation of targeted therapies for paediatric cancers of greatest unmet need; and provide a platform for data sharing to foster collaborative and hypothesis-driven research needed to catalyse clinical translation.
VPCC Childhood Cancer Model Atlas
Email VPCCcells@hudson.org.au with any enquiries.
Please note that certain materials contained in the VPCC repository may not be available immediately, may have limitations on the uses of those materials, or may require discussions between the Hudson Institute of Medical Research and the provider of those materials. Please contact Hudson if you wish to access any of the materials and Hudson will advise the relevant process.