Children’s Cancer Institute (CCI) in Sydney, Australia, is the largest paediatric cancer research organisation in the Southern Hemisphere. CCI is solely dedicated to curing childhood cancer through children cancer research, with world-leading expertise in a variety of paediatric cancers, including neuroblastoma, sarcoma, blood cancers and brain cancer.
Why work with us
All childhood cancers suffer from high unmet need in effective treatments and access to clinical trials. CCI is uniquely placed to assist companies to produce preclinical pediatric cancer data for proprietary cancer therapeutics for Pediatric Study Plan submissions to the regulators, as mandated by the recent changes in the legislation in the US and EU. Examples of innovative programs at CCI include: novel target and covalent ligand discovery, functional characterization of novel molecular drivers (using gene editing techniques), RNA-splicing modulation, target validation using an extensive range of in vitro and in vivo bioresources and multi-omics data from >2,500 patients linked to clinical outcomes.
We are employing new approaches to drug the “undruggable”, leveraging external and in-house datasets, functional genomics, chemo-proteomic drug discovery platform, computational drug discovery approaches and an internal drug screening and development pipeline.
In a long-standing partnership with the Kids Cancer Centre at Sydney Children’s Hospital, CCI co-leads the world-leading, national precision medicine program for all children with cancer, called the ZERO Childhood Cancer Program (>2500 children enrolled to date, from all 9 centers across the country) . The program has produced one of the largest pediatric cancer multi-omic real-world precision medicine datasets and biospecimen collections linked to clinical outcomes. We perform comprehensive molecular profiling (whole genome and transcriptome sequencing, methylation), liquid biopsy monitoring, and preclinical analyses (high-throughput drug screening, PDX drug testing) of patient samples to determine the genetic drivers of each child’s tumor and issue personalized treatment recommendations.
The ZERO program has demonstrated extraordinary clinical benefit compared to standard treatment approaches (Wong et al, Nature Medicine 2024) and, being the national program, it brings together all 9 pediatric oncology treatment centers and a network of national and international research partners.
