Unraveling the genetic mysteries behind childhood brain tumors is at the heart of the mission of the Center for Data Driven Discovery in Biomedicine (D3b) at the Children’s Hospital of Philadelphia (CHOP). The Children’s Brain Tumor Network (CBTN), a multi-institutional brain tumor research program whose surgery center is located in D3b, has collected tumor samples from patients around the world. Through funding from the National Cancer Institute (NCI) Childhood Cancer Data Initiative, part of the National Institutes of Health (NIH), D3b will gain molecular characterization for thousands of these brain tumor samples, providing an unprecedented level of insight into devastating cancers and cancers. paving the way for future therapeutic interventions.
Rather than a traditional monetary grant, the X01Sequencing & Genotyping Resource Access program provides critical NIH-supported resources that researchers need to conduct their research. This program supports the molecular characterization of more than 3,000 germline samples and more than 1,500 tumor samples from all pediatric brain tumor types collected by D3b through CBTN since 2011. This characterization process will prioritize the most aggressive tumor types, to support research for those cancers where there is a lack of existing data. Prior to the X01 program, approximately 1,000 of the more than 4,200 patient-derived specimens within the CBTN biobank housed in D3b have been characterized since 2017.
With molecular data coming from just a quarter of the research participants who have donated brain tumor tissue to date, CBTN has been able to support the launch of more than 150 data science-based studies. Now, with full genome sequencing available for this entire cohort, the potential for new insights into the biology of childhood brain cancer cannot be overstated.”
Adam Resnick, PhD, co-director of the D3b Center at CHOP and scientific co-chair of CBTN
Because childhood brain tumors are not common enough for a single research center to meet enough patients with the same diagnosis to collect the amount of samples or data needed to conduct substantive research, collaborations such as CBTN play a critical role in accelerating the pace. of progress. The more data available to researchers, the greater the chance of new insights leading to breakthroughs in treatment.
“Children brain tumors represent some of the most challenging targets for biomedical research, due to the scarcity of molecular data available to make useful discoveries,” said Jay Storm, MD, co-director of the D3b Center, chief of the Division of Neurosurgery. at CHOP and a CBTN principal investigator. “Through this collaboration, the NCI/NIH and the Childhood Cancer Data Initiative have enabled a brand new era in research.”
Data from this cohort of samples — generously provided by patients and their families over several years — will be made available alongside other cohorts collected as part of NCI initiatives, including Therapeutically Applicable Research to Generate Effective Treatments (TARGET) and Pediatric MATCH ( Molecular Analysis for Therapy Choice), as well as the Gabriella Miller Kids First Pediatric Research Program (Kids First) of the NIH Common Fund. Kids First is a partnership of pediatric research aimed at understanding the genetic causes and links between childhood cancer and structural birth defects. In addition, approximately 300 CBTN samples from diffuse midline glioma (DMG) and atypical teratoid rhabdoid tumor (ATRT) – two tumors affecting the central nervous system and the brain – will be characterized through a new collaboration with the Count Me In Initiative.
The sequencing for this project is supported by the NCI Childhood Cancer Data initiative under price number 1X01CA267587-01, and will be conducted at the Broad Institute of Massachusetts Institute of Technology and Harvard University.
Children’s Hospital of Philadelphia