Harnessing Data to Accelerate Discovery and Treatment of Brain Tumors

By Gerri Trooskin, Director of Partnerships, Center for Data Driven Discovery in Biomedicine.

Brain tumors are the leading cause of disease-related death in children around the world. Existing non-targeted chemotherapies and radiation - while at times effective - often leave lifelong health burdens and may cause secondary illnesses. There is an urgent need to improve therapies through targeted drugs for these children, and collaboration is key since individual hospitals have insufficient samples or information to drive advancement.

CHOP’s Center for Data Driven Discovery (D3b), led by Jay Storm, MD, Adam Resnick, PhD, and Jena Lilly, MS, is dedicated to removing barriers to research within the Neuroscience Center and throughout the entire scientific community. By breaking down silos and fostering collaboration, D3b is empowering and facilitating researchers everywhere to accelerate translational impact from the bench to the bedside for the next breakthrough. Tasks that once took researchers years to complete can now be completed in minutes, made possible through D3b’s research platforms and tools.

The Children’s Brain Tumor Network (CBTN), housed within the D3b, was formed a decade ago on a simple concept: By working together to collect and share resources, we can save more children from brain tumors. What began as only four sites devoted to data sharing in 2013 has expanded to a global consortium of researchers, clinicians, and patients across 32+ institutions worldwide who together are establishing a new standard of care for patients by sharing data, tissue samples, and expertise to accelerate discoveries.

Since its formation, the CBTN has:

• created the first clinically annotated biorepository with real-time query abilities
• made resources available to researchers across the globe — at no cost — which has shortened research time by up to 20 years
• created the Pediatric Brain Tumor Atlas (PBTA)

Now, with data generated from its newest initiative — Project Accelerate — CBTN is poised to dramatically advance the pace of translational research and the discovery of new treatments for children and young adults with brain tumors.

In 2022, the first year of Project Accelerate, the team undertook a massive data generation effort, working at capacity to extract and prepare 7,200+ tissue samples donated from patients and ship them to a sequencing center for molecular characterization. The cost of this was covered by an award from the National Institutes of Health (NIH) Kids First Research Program and the National Cancer Institute’s Childhood Cancer Data Initiative (CCDI). The team was then tasked with ensuring that all of the data that returned from the sequencing center could connect to support research — regardless of where files were created or the diversity of data types.

The project team is now working to process and analyze this priceless collection of genomic data and will integrate it within the PBTA dataset, with the goal of making it available on open access portals as quickly as possible. This data will significantly increase the size and scale of the PBTA, quadrupling the information currently available about pediatric brain tumors. All PBTA data will be available through NIH’s database of genotypes and phenotypes in the coming year, allowing easy access to researchers anywhere in the world. 

The CBTN team’s hope is that super-charging this massive dataset and optimizing it to be completely searchable by investigators at any institution, at any career level, in any research discipline will empower translational research for scientists worldwide with the goal of making precision treatments a reality for children with brain tumors in the years ahead.

D3b is now initiating a similar project focused on epilepsy using the best practices from Project Accelerate for a similar project with Pediatric Neurosurgeon Ben Kennedy, MD, and Pediatric Neurologist Ingo Helbig, MD.