National Brain Tumor Society sees a critical need to advance the field of pediatric brain tumor research. And, we believe it is essential to fill the gap in the foundational knowledge of the pediatric brain tumor field to ensure future investigations into specific pediatric tumor types are more systematic, more efficient, and yield viable treatments most effectively.

We believe the most effective route to greater understanding of pediatric brain tumors is to force a convergence of two key research areas: comprehensive molecular profiling and developmental neurobiology.

Comprehensive molecular profiling of specific tumors identifies biological targets such as genes that allow for interventions, including targeted drug therapies that will be effective for those specific tumor types. We also believe that comprehensive molecular profiling is transforming the research landscape for some tumor types. Although it is not without challenges, including sufficient collection of high-quality tumor and matched normal tissue samples, use of cutting-edge profiling technologies and how to best use advanced computational skills may yield promising results.

Molecular Profiling: Current Research & Programs

  • Genome-wide profiling of the childhood brain stem tumor diffuse intrinsic pontine glioma (DIPG). The knowledge gained from this study allows researchers to screen for possible benefits from therapeutic agents already in use for other adult cancers, which in turn may expedite clinical trials for pediatric use.
  • Genetic characterization of cell adhesion pathways in childhood supra-tentorial primitive neuroectodermal (sPNET) tumors. Cell adhesion properties affect the ability of tumor cells to migrate to parts of the central nervous system.
  • Mouse models of altered genes and pathways that cause cancer, which illustrate how combination therapies can be directed at one or the altered proteins and signaling pathways for glioma and medulloblastoma.
  • Identification of genes that are mutated in DIPG to better understand how to develop therapies that target those mutations directly.
  • Mapping the pathways that drive development of the atypical teratoid rhabdoid (AT/RT) tumor. Identifying specific mutations can point the way toward specific pathways to target with drug therapy.

Developmental neurobiology is essential to understanding normal brain developmental processes and how these relate to tumor formation and growth. Understanding the genetic, molecular, and cellular mechanisms of normal brain development is central to understanding brain tumor development. Together, studies in comprehensive molecular profiling and developmental neurobiology will result in a much more complete picture of the biological targets on which to focus in drug discovery, development, and clinical trials. This essential groundwork will enable future research into specific pediatric brain tumors to be much more focused and efficient. It will also speed the development of treatments to effectively fight tumors with minimal adverse affects to continuing normal brain development so critical for pediatric patients.

Although this is a paradigm shift for pediatric brain tumor research, we are committed to leading this adoption, through our own funding initiatives, and through our peer-to-peer advocacy within the scientific community and other brain tumor research funders.

Developmental Neurobiology: Current Research & Programs

  • Genetic targeting of cerebellar stem cells to study how tumors develop.
  • Identification of genes and cell behaviors regulated by signaling pathways in the cell of origin of a major subtype of medulloblastoma using novel genetic tools in a mouse model.
  • Analysis of the potential effects that molecularly targeted therapies could have on the niches of the brain where neurons are still developing after a child is born. The intent is to ensure that therapies don’t interfere with normal learning, memory, and development.