CHOP-Led Network Receives NIH-Supported Resources to Uncover Mysteries Behind Pediatric Brain Tumors

Unraveling the genetic mysteries behind pediatric brain tumors is at the heart of the mission of the Center for Data Driven Discovery in Biomedicine (D3b) at Children’s Hospital of Philadelphia (CHOP). The Children’s Brain Tumor Network (CBTN), a multi-institutional brain tumor research program headquartered at D3b, has collected tumor samples from patients around the world. With funding from the National Cancer Institute (NCI) Childhood Cancer Data Initiative, part of the National Institutes of Health (NIH), D3b will provide the molecular characterization of thousands of these brain tumor samples, offering an unprecedented level of knowledge on devastating cancers and paving the way for future therapeutic interventions.

Instead of a traditional cash grant, the X01 Sequencing and Genotyping Resource Access Program provides essential NIH-backed resources that are needed by researchers to conduct their research. This program will support the molecular characterization of over 3,000 germ line samples and over 1,500 tumor samples from all types of pediatric brain tumors that have been collected by D3b via CBTN since 2011. This characterization process will provide the priority to the most aggressive types of tumors, supporting research for these. cancers for which there is a lack of data. Prior to the X01 program, approximately 1,000 of the more than 4,200 patient-derived samples from the CBTN biobank hosted at D3b have been characterized since 2017.

With molecular data derived from only a quarter of 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 surveys. Now, with whole genome sequencing available for this entire cohort, the potential for new insight 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

Since 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 like CBTN plays a crucial role in accelerating the pace of progress. The more data researchers have, the greater the potential for new knowledge leading to breakthroughs in treatment.

“Childhood brain tumors represent some of the most difficult targets for biomedical research, due to the paucity of molecular data available to make actionable discoveries,” said Jay Storm, MD, co-director of the D3b Center, division head of neurosurgery at CHOP and principal investigator at CBTN. “Through this partnership, the NCI / NIH and the Childhood Cancer Data Initiative have made possible a whole new era of research. “

Data from this cohort of samples – generously provided by patients and their families over several years – will be made available alongside other cohorts that have been collected through NCI initiatives, including therapeutically applicable research for generate effective treatments (TARGET) and pediatric MATCH (Molecular Analysis for Therapy Choice), as well as the NIH Common Fund’s Gabriella Miller Kids First pediatric research program (Kids First). Kids First is a collaborative pediatric research effort to understand the genetic causes and links between childhood cancer and structural birth defects. In addition, approximately 300 diffuse midline glioma (DMG) and atypical teratoid rhabdoid tumor (ATRT) CBTN samples – two tumors that affect the central nervous system and the brain – will be characterized through a new partnership with the initiative. Count Me In.

Sequencing for this project is supported by the NCI Childhood Cancer Data initiative under award number 1X01CA267587-01, and will be performed at the Broad Institute of the Massachusetts Institute of Technology and Harvard University.