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项目摘要/摘要 儿科脑瘤联合会(PBTC)的主要目标是快速有效地 有助于对儿童中枢神经系统肿瘤的认识和治疗 通过创新、多学科和多机构的前瞻性临床试验。PBTC临床 试验的发展不仅基于已知的预后因素，而且还基于不断演变的 对儿科脑肿瘤生物学的认识。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、免疫学和 基因组学研究，努力定义和整合这些模式，以便更好地理解 儿童中枢神经系统肿瘤。这些目标将通过结束和继续实现 与成员机构的基础和翻译科学家以及临床医生建立伙伴关系 审判专家。此外，PBTC欢迎与外部调查人员建立合作伙伴关系 为创新和高度优先事项提供科学、实验室和监管支持的联盟 临床试验。PBTC将继续保持和发展与国家 癌症研究所(NCI)及其庞大的成人和儿科小组网络以及 制药和生物技术公司。PBTC最重要的目标之一是 成功完成新药和新药治疗的I期和II期临床试验 然后可以转化为监督的大型预期第二阶段和第三阶段研究的方法 儿童肿瘤学小组(COG)。最重要的长期目标是提高存活率 诊断为中枢神经系统肿瘤的儿童的预后和生活质量 发病率和死亡率，如弥漫性桥脑胶质瘤、高级别胶质瘤、婴儿 胚胎肿瘤、难治性髓母细胞瘤、室管膜瘤和低级别胶质瘤。对这件事 最后，PBTC将继续专注于通过以下途径推出新型分子靶向药物 评估肿瘤和代理组织中靶标存在的创新试验，评估 该药物下调其信号靶点的能力，并更好地关联药代动力学和 具有毒性和临床反应的药效学终点。PBTC也将继续 通过免疫治疗探索儿童脑肿瘤的治疗机会 通过干预措施以及通过在当地提供治疗。通过专注于最常见的固体 儿童肿瘤，也是#年与癌症相关的死亡人数最多的原因 儿科，PBTC将继续支持NCI帮助所有人活得更长的使命， 更健康的生活通过它对癌症研究和更好的治疗方法的确定做出贡献。

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.