Cancer kills more children than any other disease, and brain cancer is the second-leading cause of cancer-related deaths in children under age 20. With September as Childhood Cancer Awareness Month, the National Brain Tumor Society would like to emphasize our ongoing commitment to pediatric brain tumor research by raising awareness of this special month, while continuing our work every day.
For years, we have been driving research forward in the area of pediatric brain tumors. Our mission is to find better treatments, and ultimately a cure, for people living with a brain tumor today, and anyone who will be diagnosed tomorrow. We fund the research approaches that offer the most compelling results in the near term, as well as the approaches that establish a foundation for future advances.
We believe it is essential to fill the gap in the foundational knowledge of the pediatric brain tumor field, so that future investigations are more systematic, more efficient, and yield viable treatments in the most effective ways. We believe the work in comprehensive molecular profiling is transforming the research landscape for some tumor types. Our funded research in this area includes:
- Genome-wide profiling of the rare but deadly 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 to expedite new clinical trials for children.
- 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. The models show how combination therapies can be directed at one of the altered proteins and signaling pathways for glioma and medulloblastoma.
- 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. This essential groundwork will speed the development of treatments to effectively fight tumors with minimal adverse affects to continuing normal brain development so critical for pediatric patients. Our funded research in this area includes:
- 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 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.
This paradigm shift promises to result in many advancements going forward. We remain committed to this effort, both through our funding initiatives and our integrated in public policy and advocacy. Ultimately, we to find a cure, but until then we want to see children successfully treated for cancerous or benign brain tumors without suffering adverse effects so they can go on to live full and active lives.
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