The primary goal of the Pediatric Brain Tumor Consortium (PBTC) is to rapidly and effectively contribute to the understanding and cure of pediatric central nervous system (CNS) tumors through innovative, multidisciplinary and multi-institutional prospective clinical trials. PBTC clinical trial development is not only based upon known prognostic factors but also upon an evolving understanding of pediatric brain tumor biology. The PBTC will continue to utilize innovative study designs and analyses based on state-of-the-art statistical science. Novel clinical trials will incorporate relevant correlative pharmacology, biology, neuroimaging, immunologic and genomics studies in an effort to define and integrate these modalities into a better understanding of pediatric CNS tumors. These goals will be accomplished through a close and continued partnership with the member institutions' basic and translational scientists as well as the clinical trial experts. In addition, the PBTC welcomes partnerships with investigators outside the consortium providing scientific, laboratory and regulatory support for innovative and high priority clinical trials. The PBTC will continue to maintain and grow its relationship with the National Cancer Institute (NCI) and its large network of adult and pediatric groups as well as with pharmaceutical and biotechnology companies. One of the most important goals of the PBTC is to successfully complete focused phase I and II clinical trials of novel agents and treatment approaches that can be then be translated into large prospective phase II and III studies overseen by the Children's Oncology Group (COG). The overarching long-term goal is to improve survival outcome and quality of life for children with CNS tumor diagnoses that so often lead to high morbidity and mortality such as diffuse intrinsic pontine gliomas, high-grade gliomas, infant embryonal tumors, refractory medulloblastoma, ependymoma, and low-grade gliomas. To this end, the PBTC will continue to focus upon introducing novel molecularly-targeted agents via innovative trials to assess the presence of the target in tumor and surrogate tissue, evaluate the ability of the agent to down-regulate its signaling target and better correlate pharmacokinetic and pharmacodynamic endpoints with toxicities and clinical responses. The PBTC will also continue to explore therapeutic opportunities for pediatric brain tumors through immunotherapeutic interventions as well as via local delivery of treatments. By focusing on the most common solid tumor in children which also accounts for the highest number of cancer-related deaths in pediatrics, the PBTC will continue to support the NCI's mission of helping all people live longer, healthier lives through its contributions to cancer research and identification of better treatments.

Public Health Relevance

The Pediatric Brain Tumor Consortium (PBTC) conducts novel and innovative clinical trials for Pediatric Central Nervous System (CNS) malignancies that incorporate correlative biology, genomics, pharmacology and sophisticated imaging techniques to advance the understanding of high-risk and recurrent pediatric brain tumors. PBTC trials are designed to define appropriate dosing, describe pediatric-specific toxicities and tumor biology as well as to identify early signals of efficacy, thereby directly impacting future prognostication and treatment strategies. It is only through this continued prospective and systematic drug development via early phase clinical trials that the overarching goal of achieving longer, healthier lives with less morbidity can ultimately be realized for children with CNS tumors.

National Institute of Health (NIH)
National Cancer Institute (NCI)
Research Project with Complex Structure Cooperative Agreement (UM1)
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Study Section
Special Emphasis Panel (ZCA1)
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Smith, Malcolm M
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St. Jude Children's Research Hospital
United States
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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
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
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
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

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