Pediatric Brain Tumor ConsortiumA multi-institutional consortium devoted to novel phase I and II clinical evaluations of experimental treatment approaches for pediatric CNS tumors.

PBTC项目概要/摘要 儿科脑肿瘤联盟（PBTC）的主要目标是快速有效地 有助于了解和治疗小儿中枢神经系统（CNS）肿瘤 通过创新、多学科和多机构的前瞻性临床试验。PBTC临床 试验开发不仅基于已知的预后因素， 了解小儿脑肿瘤生物学。PBTC将继续利用创新研究 基于最先进的统计科学进行设计和分析。新的临床试验将 结合相关药理学、生物学、神经影像学、免疫学和 基因组学研究，努力定义和整合这些模式，以更好地了解 小儿中枢神经系统肿瘤的研究这些目标将通过一个密切和持续的 与成员机构的基础和转化科学家以及临床 审判专家此外，PBTC欢迎与调查机构以外的调查人员建立伙伴关系。 为创新和高度优先事项提供科学、实验室和监管支持的联合体 临床试验PBTC将继续保持和发展与国家的关系 癌症研究所（NCI）及其成人和儿科团体的大型网络，以及与 制药和生物技术公司。PBTC最重要的目标之一是 成功完成新药和治疗的重点I期和II期临床试验 这些方法可以转化为大型前瞻性II期和III期研究， 儿童肿瘤组（COG）首要的长期目标是提高生存率 结果和生活质量的儿童中枢神经系统肿瘤诊断，所以往往导致高 发病率和死亡率，如弥漫性内在脑桥胶质瘤、高级别胶质瘤、婴儿 胚胎性肿瘤、难治性髓母细胞瘤、室管膜瘤和低级别神经胶质瘤。本 最后，PBTC将继续专注于通过以下途径引入新型分子靶向药物： 创新性试验，以评估肿瘤和替代组织中靶点的存在， 该试剂下调其信号传导靶标的能力和更好地关联药代动力学和 药效学终点与毒性和临床反应。PBTC还将继续 探索通过免疫疗法治疗小儿脑肿瘤的机会 干预措施以及通过当地提供治疗。通过关注最常见的固体 儿童肿瘤也是癌症相关死亡人数最多的原因， 儿科，PBTC将继续支持NCI的使命，帮助所有人活得更长， 通过其对癌症研究和确定更好的治疗方法的贡献，使人们的生活更健康。

PBTC Project Summary/Abstract 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.