Children’s Hospital of Philadelphia’s Center for Data Driven Discovery in Biomedicine Receives Nearly $10 Million DHS Grant to Create Tool for Sharing Data on Cancer and Rare Diseases

“Timing is everything.”

When a child or young adult is admitted with symptoms of a brain tumor, the race begins to correctly identify, treat, and reduce the effects of each patient’s diagnosis.

Member institutions of the Children’s Brain Tumor Network (CBTN) across the globe are joining together to accelerate the discovery of new and improved therapies for affected young patients.

At each of the 33 participating CBTN primary member institutions, families of brain tumor patients are informed and consented about donating specimen samples, including tumor tissue, blood, or cerebrospinal fluid. Donated tissue that is designated for research purposes is always excess tissue not utilized by the pathology team for a diagnosis and would otherwise be discarded.

Donated research tissue is often flash-frozen using liquid nitrogen to allow it to be safely stored and transported from the collection site to the CBTN operations center. Several CBTN member sites are also equipped to process tissue immediately after the time of removal, which may enable the development of tumor models and organoids. These tumor models can recreate certain tumor characteristics for further testing and analysis. Post-mortem tissue, or tissue collected after a child’s passing, may also be collected at CBTN member sites, which may provide a wealth of information about a tumor’s progression over time, invaluable to helping researchers develop better therapeutics.

The precious specimens donated by patients play a foundational role in understanding the underlying biology of childhood brain and central nervous system tumors. While more than 100 known brain tumor subtypes have been identified to date, some hospitals may only treat a few cases of a particular subtype each year. CBTN collects and centralizes these tissue resources in larger numbers than could be collected by a single institution. Access to more significant numbers of samples empowers researchers to better understand brain tumor biology and advances science on behalf of all patients. Samples provide the most significant potential impact once the exhaustible tissue resources are converted into reusable data.

Each brain tumor tissue sample is deidentified before undergoing genetic sequencing, a process that converts the samples into data to provide the greatest value to researchers worldwide while protecting the privacy of each child. No single institution can collect enough samples on its own, making collaboration through CBTN an essential step towards more effective treatments for children.

The Impact of Working Together

Two institutions in St. Louis are working together as one CBTN member site, thanks to the close partnership between St. Louis Children’s Hospital (SLCH) and Washington University School of Medicine (WashU Medicine). WashU Medicine pediatric specialists make up the medical staff at SLCH. Pediatric oncology patients receive care through Siteman Kids at St. Louis Children’s Hospital. Siteman Kids is an affiliation with Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine.

WashU Medicine and SLCH both benefit from a shared, clinically annotated brain tumor banking program, with core facilities and expertise in high-throughput drug screening, animal imaging, and genomic sciences.

SLCH initially joined CBTN as a satellite member institution, contributing annotated clinical and genomic data from SLCH and supporting tissue collection and shipment through their affiliation at WashU Medicine.

Regional partnerships between affiliate institutions like in St. Louis are evident at other CBTN institutions, where children’s hospitals may share resources or affiliations with large university hospitals or academic research centers. These dual appointments allow for children’s hospitals to provide specialized pediatric care for children while maintaining a strong connection to adult care centers and resources from large, networked university hospitals. Shared facilities for specialized care may be available to foster collaboration and empower new innovations and treatment opportunities.

At SLCH and WashU Medicine, collaborative lab-based and clinical research efforts are coordinated through monthly group meetings focused on genomics and epigenomics, stem cell biology, experimental imaging, and mechanisms of therapeutic response and resistance in pediatric brain tumors.

Regional partnerships fuel new research breakthroughs and drive international impact to patients across the globe. Working together across institutions allows the CBTN to share valuable data and resources with hundreds of researchers beyond the scope of a single institution or health network.

“WashU Medicine and Siteman Kids at St. Louis Children’s Hospital are thrilled to join CBTN as a member institution and looks forward to partnering with CBTN to accelerate care for children diagnosed with brain and central nervous tumors. Together with CBTN, WashU Medicine and Siteman Kids at St. Louis Children’s Hospital seek to develop new and innovative treatments to understand brain tumor biology better,” said Andrew Cluster, MD, Associate Professor of Pediatrics, Hematology & Oncology at WashU Medicine.

Brain tumors remain the leading cause of disease-related death in children, but through the selfless contributions of brain tumor patients and their families, combined with the tireless dedication of professional partnerships like those cultivated at WashU Medicine and Siteman Kids at St. Louis Children’s Hospital, CBTN is pioneering new ways to detect and treat these conditions.