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Brain tumors are the leading cause of cancer-related deaths in children, and therapies are few and cause devastating long-term side effects. Genomics and epigenomics have revealed that these tumors are molecularly diverse and appear to be driven by mutations in various signaling pathways, which may present new opportunities for a more targeted therapeutic intervention. In this Review, which contains three figures and 224 references, we discuss our current understanding of the molecular basis of the three most common malignant pediatric brain tumors (medulloblastoma, ependymoma, and high-grade glioma) and the prospects for developing safer and more effective therapies.
Brain tumors are among the leading causes of cancer-related deaths in children. Although surgery, aggressive radiation, and chemotherapy have improved outcomes, many patients still die of their disease. Moreover, those who survive often suffer devastating long-term side effects from the therapies. A greater understanding of the molecular underpinnings of these diseases will drive the development of new therapeutic approaches. Advances in genomics and epigenomics have provided unprecedented insight into the molecular diversity of these diseases and, in several cases, have revealed key genes and signaling pathways that drive tumor growth. These not only serve as potential therapeutic targets but also have facilitated the creation of animal models that faithfully recapitulate the human disease for preclinical studies. In this Review, we discuss recent progress in understanding the molecular basis of the three most common malignant pediatric brain tumors—medulloblastoma, ependymoma, and high-grade glioma—and the implications for development of safer and more effective therapies.