Breaking Down Barriers to Transform Pediatric Brain Tumor Research

When it comes to childhood brain tumor research, the idea of collaboration is not just an idealistic concept; it's quickly becoming a lifeline. Doctors and scientists who study children's brain tumors often work on their own. This means they keep their important information private, which slows down the search for new treatments. However, a new partnership between the Children’s Brain Tumor Network (CBTN) and Day One Biopharmaceuticals (Day One) is transforming the way researchers and for-profit businesses collaborate for the common good.

This powerful alignment aims to accelerate the development of new therapies for childhood brain tumors by leveraging the massive and well-curated database housed by CBTN to support Day One's drug development efforts. It's a prime example of how industry can work with CBTN to directly influence the drug development pipeline.

The Power of Collaboration and Comprehensive Data

The core of this cooperation lies in sharing comprehensive, consented pediatric patient data. Day One recognized the unique way CBTN has its information set up, allowing for an external, real-world grouping of patients. Unlike limited patient data collections, the CBTN ecosystem data lets scientists and researchers ask new questions from a variety of angles, including clinical, imaging, and biological data.

The partnership began with a set of 50 patient record sets that include clinical, molecular, and imaging data. The success of this initial effort prompted the second phase, which provides for records from an additional 200 pediatric and young adults with BRAF-altered low-grade gliomas (pLGG).

A comprehensive approach is crucial because response to treatment is defined by medical and imaging observations. The ability of CBTN to provide a research-based repository of real-world MRI images allows for a more objective and centralized review of patient data. CBTN is the first organization to hold such a repository.

The clinical dataset delivered by the Biospecimen and Clinical Research Unit (BCRU) and Advanced Data Applications and Platform Technologies Unit (ADAPT) teams at the Center for Data-Driven Discovery in Biomedicine (D3b), the operations hub of CBTN, has been hailed as an example of the dedication, expertise, and collaboration across CBTN. The quality of the deliverables was exactly what Day One had hoped for and was described as the highest quality they had seen. This success is not just a win for this specific project; it also proves the caliber of work CBTN can deliver.

“This partnership represents a new model for collaboration between a pharmaceutical entity and a research organization,” said Dr. Angela Waanders, Section Head of NeuroOncology at the Ann & Robert H Lurie Children’s Hospital of Chicago. “It's the first time an international consortium like CBTN has been able to provide this type of external, real-world research data that includes not only clinical information but also research-derived data from additional testing and MRI images.”

Accelerating Discovery and Improving Outcomes

By leveraging data from CBTN, the project could significantly reduce the time it takes to get drugs to patients, and importantly, it may reveal treatments and therapies with fewer long-term side effects.

Data that may otherwise take a traditional clinical trial five years or more to gather, CBTN can provide much more quickly. This approach also helps improve the process of extracting and organizing data from electronic health records, laying the foundation for AI and machine learning to make future research even more efficient.

A key aspect of this collaboration is its focus on a genetically defined patient group, which is crucial for testing newer, more targeted drugs, such as Day One’s pan-RAF inhibitor. This is an important distinction, as the drugs previously prescribed for this type of cancer could actually worsen the condition.

This shared approach also helps to identify drugs that don't work, which is equally important. It provides a more objective way to determine if a treatment is truly effective and can help clinicians identify potential biases in their approach. This is particularly relevant for rare tumors, where every data point is critical.

Building a Sustainable Future

This partnership is not just a one-off project; it's a model for the future. The data CBTN collects for this collaboration becomes part of the CBTN Pediatric Brain Tumor Atlas (PBTA) and will be available to researchers worldwide. This provides CBTN with additional resources to expand its scope of work and further enrich its clinical data. As Dr. Waanders stated, “This is a culmination of a vision held as far back as 2012: to operationalize and optimize the ability to collect and share comprehensive data within the CBTN ecosystem so that it can be scaled more broadly.”

Early success of this partnership between CBTN and Day One demonstrates that by breaking down silos and embracing a new paradigm of shared, open data, scientists can build a sustainable system that drives innovation for generations to come. It proves that when foundations like CBTN, pediatric researchers, and innovative corporations collaborate, they can bring more than hope to families and lay the groundwork for further breakthroughs in cancer research.