Pediatric brain tumors: Improving survival through integration of clinical molecular diagnostics and biologically targeted therapies into clinical trials

项目总结/摘要： 中枢神经系统（CNS）肿瘤是儿童癌症死亡的主要原因。其中许多 存活下来的患者由于治疗的毒性而遭受显著的终身发病率。迫切需要 提高儿童脑肿瘤患者生存率并降低治疗毒性。我的研究重点是提高 通过开发新的治疗方法和临床试验， 对疾病分子驱动力的生物学见解。我通过合作研究来进行研究 国家临床试验网络（NCTN），特别是儿童肿瘤学， 组（COG）。在全国范围内，我担任COG CNS委员会的临床副主席， NIH脑肿瘤指导委员会（BMSC）的COG代表和 美国国立卫生研究院（NIH）资助的儿科早期临床试验网络的药物优先委员会（APC） （PEP-CTN）。我最近还领导创建了一个名为INSPiRE联盟的数据共享计划 旨在促进临床试验和基因组数据共享之间的COG和其他国际 临床试验联盟和登记处。在当地，我担任西雅图儿童医院网站的主要负责人， 西雅图儿童本汤儿童癌症中心临床研究医学主任 研究，西雅图儿童肿瘤银行和生物学研究的首席研究员，和医学 儿科脑肿瘤项目主任。这些相辅相成的国家和地方作用使我能够 为广泛的转化和临床研究工作提供支持，提供所有阶段的 为西雅图的儿童提供研究，并将西雅图的研究发现翻译给世界各地的儿童 通过合作临床试验的发展。该奖项提供的机会将允许 我将投入大量时间来加速高影响力临床试验的发展，并支持关键的 努力改善分子和临床数据共享以及当地获得试验的机会。

PROJECT SUMMARY/ABSTRACT: Tumors of the central nervous system (CNS) are the leading cause of cancer death in children. Many of those who do survive suffer from significant lifelong morbidity due to toxicity of therapy. There is an urgent need to improve survival and decrease toxicity of therapy in children with brain tumors. My research focus is to improve survival for children with CNS tumors through the development of novel therapeutics and clinical trials based on biologic insights into the molecular drivers of disease. I conduct my research through collaborative studies that are made possible by the National Clinical Trials Network (NCTN), specifically the Children’s Oncology Group (COG). Nationally, I serve in a leadership role as the clinical vice-chair of the COG CNS committee, COG representative to the NIH Brain Malignancies Steering Committee (BMSC), and CNS representative to the Agent Prioritization Committee (APC) of the NIH-funded Pediatric Early Phase Clinical Trials Network (PEP-CTN). I have also recently led the creation of a data-sharing initiative called the INSPiRE consortium which aims to facilitate clinical trials and genomic data sharing between the COG and other international clinical trials consortia and registries. Locally, I serve as the Seattle Children’s Hospital site PI for COG, Medical Director of Clinical Research for the Seattle Children’s Ben Towne Center for Childhood Cancer Research, Principal Investigator of the Seattle Children’s Tumor Banking and Biology Study, and Medical Director of the Pediatric Brain Tumor Program. These complementary national and local roles allow me to provide support to a wide range of translational and clinical research efforts, providing access to all phases of research to children in Seattle and translating research discovery in Seattle to children across the world through the development of collaborative clinical trials. The opportunities afforded by this award would allow me to devote significant time to accelerate the development of high-impact clinical trials and to support critical efforts to improve molecular and clinical data sharing and local access to trials.