#BrainTumorAwarenessMonth: Increased Support of Collaborative Brain Tumor Research is Critical to Improving Treatments and Discovering Cures for Children

*Note: This article comes courtesy of the Children’s Brain Tumor Network (CBTN), for which the Center for Data Drive Discovery in Biomedicine is the operations center.  The CBTN is a collaborative, multi-institutional research program dedicated to the study and treatment of childhood brain tumors.  Learn More


Every May, foundations, patient groups, hospitals, and researchers around the world call on the public to “Go Gray in May” to raise awareness about Brain Tumors and to encourage public support for research into these often devastating and life-threatening diseases in children and adults.

According to the Central Brain Tumor Registry of the United States (CBTRUS), an estimated 86,970 new cases of primary malignant and non-malignant brain and central nervous system (CNS) tumors are expected to be diagnosed in the US by the end of 2019.  Nearly 4,000 of those new tumor diagnoses will be applied to children under the age of 14. An additional 12,290 new cases of brain and CNS tumors will be found in Adolescents and Young Adults (AYA) by the end of this year.

Brain and CNS tumors are the most prevalent form of pediatric cancer in children under age 19, and are currently the leading cause of cancer-related deaths among children and adolescents.  Yet only 4% of all federal funding for cancer research is devoted to the study of all forms of childhood cancer.

This lack of public awareness and federal support for the study of pediatric brain tumors has contributed to a woefully low amount of progress made in treatments for children over the last several decades.  In the past 30 years, only four FDA-approved drugs – and one medical device – have been developed to treat all brain tumors. There has never been a drug developed and approved to specifically treat malignant brain tumors in children.  Furthermore, some of the deadliest types of childhood brain tumors have virtually no effective treatment options for children.

Change is Coming

In the face of these daunting statistics, patients, foundations, researchers, and institutions across the country and around the globe have begun forging new and innovative ways to share knowledge and resources, support one another, collaborate, and drive pediatric brain tumor research forward to improve treatments and save the lives of children experiencing these devastating diseases.

Founded at the Children’s Hospital of Philadelphia in 2009 and launched in 2011, the Children’s Brain Tumor Network (CBTN) is a global collaborative, multi-institutional research program dedicated to the study and treatment of childhood brain tumors. As part of its research efforts, the CBTN has developed a network of informatics and data applications which allow researchers from across the world to work together in real time to discover cures.

The CBTN fosters collaboration between clinicians and researchers from 16 top-tier national and international institutions, facilitating the analysis of a broad range on childhood brain tumor biospecimens and enabling researchers across the world to share their findings toward improving treatments and outcomes for children with brain tumors.

Additionally, the Consortium has partnered with more than 40 foundations, who have raised awareness among the brain tumor patient community and the public of the CBTN’s critical work, and have thus far provided a majority of the funding that has enabled the Consortium to grow, drive research for improved standard of care, and create the open-access platforms that enable real-time global collaborations.

With this support from patients, foundations, and researchers since its launch in 2011, the CBTN has grown from one scientific project analyzing data from a collection of 300 biospecimens, to fostering more than 70 scientific biospecimen and data projects across its 16 member institutions and research community, and coordinating the storage and meticulous analysis and harmonization of nearly 40,000 biospecimens collected from over 3,000 study participants.

The CBTN is currently the largest pediatric brain tumor biorepository on Earth, complimented by highly annotated longitudinal clinical, genomic, imaging, and histology data – all available by request to researchers anywhere in the world!

Making an Impact

The foundational efforts of the CBTN are providing researchers with the resources, data, and biospecimens they need to allow them to form and test hypotheses with immediacy; significantly reducing the years of work and funding that have traditionally been required to make meaningful progress in the lab and in the clinic. Researchers can now access rich collections of “multi-omics” data, along with curated clinical/phenotypic datasets.

Through collaborative efforts with the Pacific Pediatric Neuro-Oncology Consortium (PNOC), CBTN has identified new therapeutic targets, including 4 clinical trials, for the treatment of High-grade Gliomas; among the deadliest brain tumors in children. This largest-available collection of High-grade Glioma data includes clinical, paired-omic data, and biospecimen samples, and is available through the Kids First Data Resource Portal, Cavatica, and PedcBioPortal platforms.

With the CBTN’s biospecimens and data focusing on Craniopharyngioma – a rare type of brain tumor derived from embryonic tissue – researchers are currently working to identify new targets for active clinical trials, which is leading to the development of additional, minimally-invasive surgical techniques.  These innovative developments have resulted in a new standard of care for patients by discovering innovative uses for drug therapeutics in combination with gentler surgical interventions.

Through the generation and integration of genomic and molecular data associated with Low-grade Astrocytomas and other Low-grade Gliomas (LGG), studies in the laboratory have enabled a better understand the biological mechanisms of these tumor types.  This work has led to the development of new clinical trials to improve therapeutics for children with LGGs, and is providing the first early steps toward a precision medical approach to treating these tumors.

In September of 2018, the CBTN launched its Pediatric Brain Tumor Atlas, a fully-harmonized collection of match tumor/normal, WGS, RNAseq, and miRNA-Seq data representing more than 30 different pediatric brain tumor types collected from over 1000 study participants.  Included in this dataset, which is currently the largest collection of harmonized pediatric brain tumor data on the planet, are aligned/unaligned reads, histology images, operation reports, pathology reports, gVCF, radiology images, and radiology reports – all available to researchers in real-time, with no embargo period.

Moving Forward

While the CBTN and the broader cancer research community as a whole have made great strides in recent years toward a more cooperative and fruitful model of studying and treating brain tumors, there is still much more work to be done.  Luckily, there are now more ways than ever for dedicated individuals at every level to get involved and move us all closer toward a future free of these diseases.

Data scientists, laboratory researchers, and clinicians working anywhere in the world can apply for access to CBTN biospecimens and genomic data to empower and accelerate their research and treatment efforts.

Patient families can learn how to donate tissue and other biosamples, and to contribute data toward a more complete genetic understanding of these diseases in children, taking a defiant stand against cancer and hastening the discovery of cures for future generations.

Foundations, industry, and individuals anywhere can provide philanthropic support, sustaining the advancement of brain tumor research and helping to save the lives of countless children the world over.

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