Major advances have been achieved in the discovery and application of targeted therapy for adult solid tumors, but therapeutic options for pediatric solid tumors have been limited. Current precision medicine programs focus on genomic sequencing, identifying the genes in a tumor’s DNA, to identify mutations, or changes, that may predict patients’ responses to targeted therapies. Unfortunately, currently 10% or fewer cancer patients have mutations that can be targeted and only 50% of those patients respond to therapy. This shows a need to identify new targets for treatment that improve treatment efficacy and limit the side effects of standard chemotherapy and radiation therapy. This project focuses on developing and utilizing individual patients’ tumor cells to identify new therapeutic targets and repurpose existing targets using new technologies, particularly for patients with high risk solid tumor (sarcomas, brain malignancies, refractory Wilms tumour, and neuroblastoma). Researchers will access the unique and comprehensive dataset provided by the Pediatric Brain Tumor Atlas to complete this work, and the Children’s Brain Tumor Network will also provide the computational and technological support necessary for this high level analysis.
What are the goals of this project?
Researchers will complete comprehensive molecular analysis on pediatric brain cancers and perform a screen to identify therapeutic targets.
What is the impact of this project?
Treatment efficacy for many forms of pediatric brain tumors is low and often leaves patients with lasting side effects. A more comprehensive understanding of each cancer type could lead researchers to novel therapies. Screening for therapeutics targets will potentially identify target therapeutic opportunities.
Why is the CBTN request important to this project?
In addition to access the comprehensive data provided by the Pediatric Brain Tumor Atlas, CBTN will also provide this project with the necessary computational support to complete this complex analysis.
The Children's Brain Tumor Network contributed to this project by providing access to the Pediatric Brain Tumor Atlas and by leading the computational efforts for the brain cancer analysis.
Annie Huang, Hospital for Sick Children, Canada
- Perform Assay for Transposase Accessible Chromatic with high-throughput sequencing (ATAC-seq) to chart chromatin accessibility at a genome-wide scale
Roger Daly, Monash University, Australia
- Perform global phospho-proteomic profiling to identify signatures of activated oncogenic pathways and kinases on patient tumour models grown in culture conditions.
Other team members:
- Claire Sun, Hudson Institute of Medical Research, Australia
- Daniel Gough, Hudson Institute of Medical Research, Australia
- Duncan Crombie, Hudson Institute of Medical Research, Australia
Ron Firestein, MD, PhD
Associate Professor Ron Firestein is the Head of the Cancer Centre at the Hudson Institute of Medical Research and Head of Research for the Hudson Monash Paediatric Precision Medicine Program. A/Prof Firestein is a physician scientist double board certified in Pathology (Anatomic and Molecular Genet
Hudson Institute of Medical Research
Holly Beale, PhD
Holly is an investigator at UC Santa Cruz Treehouse Childhood Cancer Initiative in Santa Cruz, California.
UC Santa Cruz Treehouse Childhood Cancer Initiative
Roger Reddel, MD, BSc (Med), MBBS, FRACP, FAAHMS, FAA
Cellular immortalisation, Cellular senescence, tumour suppressor genes, Telomeres, Proteomics
The University of Sydney
Jason Cain, PhD
Dr. Jason Cain is Head of the Developmental and Cancer Biology research group in Hudson Institute’s Centre for Cancer Research and a Chief Investigator for the Hudson Monash Paediatric Precision Medicine Program. A developmental and cancer biologist, Dr. Cain is driving cutting-edge research to unde
Hudson Institute of Medical Research
Adam Resnick, PhD
Adam Resnick is the Director of Data Driven Discovery in Biomedicine (D3b) at Children’s Hospital of Philadelphia (CHOP) responsible for leading a multidisciplinary team to build and support a scalable, patient-focused healthcare and educational discovery ecosystem on behalf of all children. He is a
Children’s Hospital of Philadelphia
Hudson Institute of Medical ResearchJoined on
A global bioscience medical research leader, Hudson Institute’s sole focus is on powering breakthrough scientific discoveries into improved healthcare that will transform lives. Our 442 scientists research four areas of medical need - inflammation, cancer, reproductive health and pregnancy, and infa
UC Santa Cruz Treehouse Childhood Cancer InitiativeJoined on
The Treehouse Childhood Cancer Initiative was established to analyze genomic data of children with cancer in the context of large data sets of both pediatric and adult cancers. This allows researchers to identify situations where an available drug is predicted to work for a given patient’s tumor. Tr
Children’s Hospital of PhiladelphiaJoined on
Operations Center for the Children’s Brain Tumor Tissue Consortium, the Children’s Hospital of Philadelphia (CHOP) is currently ranked 1st nationally for their Pediatric Cancer Program by U.S. News & World Report. CHOP’s Biobank is home to the CBTTC’s pediatric brain and CNS tumor biorepository; the