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.

Public Health Relevance

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.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Research Project--Cooperative Agreements (U01)
Project #
Application #
Study Section
Special Emphasis Panel (ZCA1-SRRB-K (J1))
Program Officer
Smith, Malcolm M
Project Start
Project End
Budget Start
Budget End
Support Year
Fiscal Year
Total Cost
Indirect Cost
St. Jude Children's Research Hospital
United States
Zip Code
Kilburn, Lindsay B; Kocak, Mehmet; Baxter, Patricia et al. (2018) A pediatric brain tumor consortium phase II trial of capecitabine rapidly disintegrating tablets with concomitant radiation therapy in children with newly diagnosed diffuse intrinsic pontine gliomas. Pediatr Blood Cancer 65:
Banerjee, Anuradha; Jakacki, Regina I; Onar-Thomas, Arzu et al. (2017) A phase I trial of the MEK inhibitor selumetinib (AZD6244) in pediatric patients with recurrent or refractory low-grade glioma: a Pediatric Brain Tumor Consortium (PBTC) study. Neuro Oncol 19:1135-1144
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
Gururangan, Sridharan; Reap, Elizabeth; Schmittling, Robert et al. (2017) Regulatory T cell subsets in patients with medulloblastoma at diagnosis and during standard irradiation and chemotherapy (PBTC N-11). Cancer Immunol Immunother 66:1589-1595
Zukotynski, Katherine A; Vajapeyam, Sridhar; Fahey, Frederic H et al. (2017) Correlation of18F-FDG PET and MRI Apparent Diffusion Coefficient Histogram Metrics with Survival in Diffuse Intrinsic Pontine Glioma: A Report from the Pediatric Brain Tumor Consortium. J Nucl Med 58:1264-1269
Poussaint, Tina Young; Vajapeyam, Sridhar; Ricci, Kelsey I et al. (2016) Apparent diffusion coefficient histogram metrics correlate with survival in diffuse intrinsic pontine glioma: a report from the Pediatric Brain Tumor Consortium. Neuro Oncol 18:725-34
Salloum, Ralph; Hummel, Trent R; Kumar, Shiva Senthil et al. (2016) A molecular biology and phase II study of imetelstat (GRN163L) in children with recurrent or refractory central nervous system malignancies: a pediatric brain tumor consortium study. J Neurooncol 129:443-451
Han, Kelong; Peyret, Thomas; Quartino, Angelica et al. (2016) Bevacizumab dosing strategy in paediatric cancer patients based on population pharmacokinetic analysis with external validation. Br J Clin Pharmacol 81:148-60
Mulkern, Robert V; Ricci, Kelsey I; Vajapeyam, Sridhar et al. (2015) Pediatric brain tumor consortium multisite assessment of apparent diffusion coefficient z-axis variation assessed with an ice-water phantom. Acad Radiol 22:363-9
Robinson, Giles W; Orr, Brent A; Wu, Gang et al. (2015) Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. J Clin Oncol 33:2646-54

Showing the most recent 10 out of 65 publications