Angela Waanders

The effective treatment of brain tumors requires a robust understanding of their biology. Using rare, high quality neurocytoma samples provided by the Children’s Brain Tumor Network, researchers seek to develop new treatments for this disease.

Embryonal tumors with multilayer rosettes (ETMR) have recently been reclassified and clinical investigations are needed to understand their occurrence and prevalence. ETMR specimens from the Pediatric Brain Tumor Atlas will be used by researchers to begin building a robust understanding and characterization of the tumor.

Data points to disparities of cancer diagnosis and outcomes across children in different racial and ethnic groups. In an effort to better understand these disparities, researchers will interrogate data provided by the Pediatric Brain Tumor Atlas.

Research into adult cancer has benefited greatly from large scale genomic research. Until now due to the limited resources and availability pediatric brain tumors have not been part of large-scale genomic generation efforts. The CBTN aims to create a comprehensive genomic atlas for use by researchers worldwide to unlock how brain tumors develop, grow, survive, and how they can be treated and cured.

Low grade gliomas are often associated with the genetic disorder neurofibromatosis type 1 and research is needed to develop targeted therapies for such tumors. The Children’s Brain Tumor Network is providing researchers with rare samples necessary to accelerate progress in the treatment of NF1-LGGs.

Studies have shown that many patients with germline mutations, mutations that can be passed down, have a predisposition to central nervous system tumors. Researchers will use data from the Pediatric Brain Tumor Atlas along with patient’s clinical data and family history to study the impacts of predisposition on patient outcomes.

In November 2020, the FDA released guidance to help promote clinical trial enrollment practices that could lead to clinical trials that better reflect diverse populations who may benefit from treatments, medical devices, or drugs that are being investigated. Despite previous efforts, there remain gaps in inclusion of under-represented populations in many clinical trials. One of the barriers to being inclusive among all groups are the languages that are offered for informed consent practices. Currently, the Children’s Hospital of Philadelphia and collaborating institutions typically offer multilingual staff and informed consents in limited languages. But, the options available at each site vary and can be costly and burdensome at the level of each individual institution/clinical research team. Our goal is to remove this potential barrier by providing consortium level informed consents and study documents in multiple languages to ensure enrollment and recruitment of under-represented minorities (URM) in languages understood by a diverse population of patients and families. By providing these necessary resources, non-English speaking patients and families will be afforded the same opportunity to understand and participate in clinical trial enrollments as English-speaking patients and families.

We have not yet discovered why the vast majority of children develop brain cancer. Utilizing CBTN data and Kids First data resources, researchers at CBTN are working to answer questions about the cause of pediatric brain tumors.

Many pediatric brain cancer patients experience toxicities related to the use of vincristine in treatment protocols. Using the Pediatric Brain Tumor Atlas, this study will help to define the frequency of genetic risks for side effects in an effort to lessen the need for treatment reductions.