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

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.

项目概要/摘要： 中枢神经系统 (CNS) 肿瘤是儿童癌症死亡的主要原因。 由于治疗的毒性而确实遭受严重的终生发病的人迫切需要。 提高脑肿瘤儿童的生存率并降低治疗的毒性。 通过开发新疗法和基于临床试验的方法来提高中枢神经系统肿瘤儿童的生存率 我通过合作研究来进行对疾病分子驱动因素的生物学见解。 国家临床试验网络 (NCTN)，特别是儿童肿瘤学使之成为可能 在全国范围内，我担任 COG CNS 委员会的临床副主席， NIH 脑恶性肿瘤指导委员会 (BMSC) 的 COG 代表和 NIH 脑恶性肿瘤指导委员会 (BMSC) 的 CNS 代表 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 COG 和其他国际组织之间的临床试验和基因组数据共享 在当地，我担任西雅图儿童医院 COG 的临床试验联盟和注册中心 PI。 西雅图儿童本汤儿童癌症中心临床研究医学主任 研究、西雅图儿童肿瘤库和生物学研究的首席研究员以及医学 儿科脑肿瘤项目主任。这些互补的国家和地方角色使我能够 为广泛的转化和临床研究工作提供支持，提供进入各个阶段的机会 针对西雅图儿童的研究并将西雅图的研究发现转化为世界各地的儿童 通过合作临床试验的发展，该奖项将提供机会。 我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 改善分子和临床数据共享工作以及本地试验的获取。