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. In translating multiple innovative therapies from the laboratory to early phase clinical testing in pediatric patients with CNS diseases, our future PBTC clinical trials will focus more on understanding genomics related to the treatment of these tumors, as well as pharmacogenomics in PBTC phase II trials. The OBC is in the process of converting the computing infrastructure that supports the PBTC to the MediData Rave system and will definitely have it in place by the time the new grant period starts. We have already begun to utilize the NCI-sponsored Cancer Trials Support Unit (CTSU) for regulatory support. Patient registration for all PBTC trials initiated in the new grant period will be via the CTSU Oncology Patient's Enrollment Network (OPEN). Thus, these changes will facilitate closer collaborations with the COG, which is using MediData Rave and the CTSU. This will facilitate providing data from PBTC trials to the COG for the implementation of subsequent trials. In addition, these systems provide a means for facilitating selected COG member institutions to participate in PBTC trials that require accrual of patients with very rare CNS tumors, as may be required for trials of new agents that target tumors with specific pathways activated or with specific genetic abnormalities. The Mission of the PBTC is to contribute rapidly and effectively to the understanding and cure of pediatric CNS tumors through innovative, multidisciplinary, multi-Institutional clinical trials based upon evolving knowledge of brain tumor biology and using study designs and analyses based on state-of-the-art statistical science. The PBTC is committed to integrating clinical trials with correlative pharmacology, biology, neuroimaging and genomics studies. PBTC investigators recognize their profound responsibility to study the special needs of their patients and families, as they face enormous challenges during and after therapy.

Public Health Relevance

Central nervous system (CNS) tumors represent 20% of childhood cancers, but are the leading cause of cancer-related childhood deaths. Five-year overall survival (OS) in children with diffuse intrinsic pontine glioma (DIPG), high-grade gliomas, (HGG), ependymoma (EP) or medulloblastoma (MB) are 10%, 20%, 60% and 70% respectively, with dismal outcome for patients with recurrent disease.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Research Project with Complex Structure Cooperative Agreement (UM1)
Project #
Application #
Study Section
Special Emphasis Panel (ZCA1-GRB-I (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
Lulla, Rishi R; Goldman, Stewart; Yamada, Tohru et al. (2016) Phase I trial of p28 (NSC745104), a non-HDM2-mediated peptide inhibitor of p53 ubiquitination in pediatric patients with recurrent or progressive central nervous system tumors: A Pediatric Brain Tumor Consortium Study. Neuro Oncol 18:1319-25
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

Showing the most recent 10 out of 24 publications