To date, one must conclude that the pace of progress in disease control and preservation of functional integrity and quality of life for pediatric brain tumors remains below that of most other major types of childhood cancers. Recent advances in cellular and molecular biology have further characterized some of the more common pediatric brain tumor histiotypes - identifying molecular targets against which therapeutic strategies are now being developed. Thus, the scientific opportunities for advancing treatment options for childhood brain tumors have expanded considerably since the inception of the Pediatric Brain Tumor Consortium (PBTC) in April 1999, as the molecular characteristics of childhood brain cancers are understood far better today. These advances are paralleled by an expansion in the number and variety of anticancer agents targeted towards specific signaling pathways that control growth and proliferation of cancer cells. The combination of both factors, increased understanding of cancer biology and availability of novel therapeutic strategies, creates clinical research opportunities for the PBTC to continue to exploit. The mission of the PBTC is to contribute rapidly and effectively to the understanding and cure of pediatric CNS tumors taking advantage of recent advances in cellular and molecular biology by using these findings to drive the conduct of multi-center, multidisciplinary innovative studies with designs and analyses based on uniformly high-quality statistical science. While the primary mission of the PBTC is to identify, through laboratory and clinical science, superior treatment strategies for children with brain cancers, the PBTC investigators recognize their profound responsibility to meet the special needs of the children and families as they face this enormous challenge and will address these needs with focused quality of life research. Members are also committed to working within their institutions and communities to improve support services and follow-up care for these patients and their families.
Brain tumors represent 20% of all childhood cancers;approximately 2700 children and adolescents presenting with CNS tumors annually in the U.S. A significant proportion has aggressive, malignant histiotypes typically with 5-year survival rates of less than 10-25%. The current treatment options for children with brain tumors result in suboptimal functional outcomes and a substantial proportion of children with brain cancers who do survive exhibit functional impediments.
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|Patel, Y T; Daryani, V M; Patel, P et al. (2017) Population Pharmacokinetics of Selumetinib and Its Metabolite N-desmethyl-selumetinib in Adult Patients With Advanced Solid Tumors and Children With Low-Grade Gliomas. CPT Pharmacometrics Syst Pharmacol 6:305-314|
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