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

儿科脑肿瘤：通过将临床分子诊断和生物靶向治疗整合到临床试验中来提高生存率

• 批准号: 10563032
• 负责人: Sarah Elisabeth Sherr Leary
• 金额: $ 17.53万
• 依托单位: SEATTLE CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-03-21 至 2028-02-29
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10563032
• 关键词: Acceleration; Award; Biological; Brain; Brain Neoplasms; Cancer Etiology; Central Nervous System; Central Nervous System Neoplasms; Child; Childhood; Childhood Brain Neoplasm; Clinical; Clinical Data; Clinical Research; Clinical Trials; Clinical Trials Network; Data; Development; Disease; Enrollment; Equity; Funding; Goals; Infrastructure; Institution; International; Leadership; Malignant Childhood Neoplasm; Malignant Neoplasms; Molecular; Morbidity - disease rate; National Clinical Trials Network; Operations Research; Pediatric Hospitals; Pediatric Oncology Group; Phase; Physician Executives; Principal Investigator; Qualifying; Registries; Research; Role; Scientist; Site; Time; Translating; Translational Research; Treatment-related toxicity; Tumor Bank; Tumor Biology; United States National Institutes of Health; Work; anticancer research; childhood cancer mortality; clinical trial implementation; data sharing; early phase clinical trial; experience; genomic data; improved; insight; molecular diagnostics; next generation; novel therapeutics; programs; risk stratification; targeted treatment; Acceleration; Award; Biological; Brain; Brain Neoplasms; Cancer Etiology; Central Nervous System; Central Nervous System Neoplasms; Child; Childhood; Childhood Brain Neoplasm; Clinical; Clinical Data; Clinical Research; Clinical Trials; Clinical Trials Network; Data; Development; Disease; Enrollment; Equity; Funding; Goals; Infrastructure; Institution; International; Leadership; Malignant Childhood Neoplasm; Malignant Neoplasms; Molecular; Morbidity - disease rate; National Clinical Trials Network; Operations Research; Pediatric Hospitals; Pediatric Oncology Group; Phase; Physician Executives; Principal Investigator; Qualifying; Registries; Research; Role; Scientist; Site; Time; Translating; Translational Research; Treatment-related toxicity; Tumor Bank; Tumor Biology; United States National Institutes of Health; Work; anticancer research; childhood cancer mortality; clinical trial implementation; data sharing; early phase clinical trial; experience; genomic data; improved; insight; molecular diagnostics; next generation; novel therapeutics; programs; risk stratification; targeted treatment

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.

项目摘要/摘要： 中枢神经系统（CNS）的肿瘤是儿童癌症死亡的主要原因。其中许多 由于治疗的毒性，生存的人患有终生发病率。迫切需要 改善脑肿瘤儿童的生存率和降低治疗的策略。我的研究重点是改善 通过开发新的治疗和基于临床试验的CNS肿瘤儿童的生存 关于疾病分子驱动因素的生物学见解。我通过协作研究进行研究 国家临床试验网络（NCTN），特别是儿童肿瘤学使这成为可能 组（COG）。在全国范围内，我担任COG CNS委员会临床副主席的领导角色， 代表NIH Brain Malignancys指导委员会（BMSC）的COG和CNS代表 NIH资助的小儿早期临床试验网络的代理优先委员会（APC） （PEP-CTN）。我最近还领导了一项名为Inspire Consortium的数据共享计划的创建 旨在促进COG与其他国际的临床试验和基因组数据共享 临床试验财团和注册表。在当地，我担任西雅图儿童医院网站PI cog， 西雅图儿童本·汤恩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主要研究者以及医学 小儿脑肿瘤计划主任。这些完整的国家和地方角色使我能够 为各种翻译和临床研究工作提供支持，从而提供了对所有阶段的访问 对西雅图的儿童进行研究，并将西雅图的研究发现转化为世界各地的儿童 通过开发协作临床试验。该奖项提供的机会将允许 我要降低大量时间来加快高影响力临床试验的发展并支持关键 改善分子和临床数据共享以及本地访问试验的努力。