Pediatric Oncology In Vivo Testing Program Coordinating Center

项目概要 NCI 儿科体内测试计划 (Ped-In Vivo-TP) 的总体目标是改善以下疾病的结果： 儿科癌症患者并满足加速治愈和公平研究的要求 (RACE) 儿童法案，旨在评估为成人开发的儿科靶向抗癌药物的功效 上下文。为了协调新的 Ped-In Vivo-TP 计划的活动，我们组建了一个团队 来自杰克逊实验室 (JAX) 和七桥基因组 (SB) 的研究人员采用独特的组合 体内癌症模型、可扩展的基于云的数据管理和分析的专业知识和经验 系统、信息学资源开发和多站点项目协调。结合互补 JAX 和 SB 的优势为协调中心最大化短期和 儿科体内测试计划对推进精准医学应用的长期影响 在儿科肿瘤学中。我们将管理全面且有凝聚力的测试计划以提高精度 通过制药公司之间有效的公私伙伴关系，在儿科肿瘤学领域开展医学研究， 监管机构、资助者和研究组织。我们将通过以下目标来实现这一目标： 目标 1：建立并维护儿科体内检测协调中心 (PIVOT CC)，以提供 为儿科体内测试计划的不同利益相关者提供行政和后勤支持 财团。我们将利用我们团队数十年的多站点项目经验 管理、癌症模型开发、体内癌症模型标准化测试、数据管理 和分析以及信息学资源开发，以确保及时决策，符合 Ped-In Vivo-TP 内的标准协议、资源跟踪和有效通信。目标 2： 提供数据管理、统计和生物信息学支持，确保数据安全和完整性。 我们将利用 JAX 和 SB 的现有协议和软件系统来识别相关的体内癌症 模型并收集、分析和安全管理从测试中心生成的数据 财团。我们将对联盟数据进行统计和生物信息学分析，以可靠地告知 评估新型治疗药物在儿科肿瘤学环境中的疗效。我们将开发一个公共—— 面向数据门户，与更广泛的科学界共享数据和方法。目标 3：提供 科学协调，以维持高效、有效的临床前测试渠道。我们会画 我们团队在体内药理学方面的丰富经验以及类似联盟的协调 Ped-In Vivo-TP 中心在流程的各个阶段管理和协调药物测试，从 识别相关体内模型以生成最终技术报告并发布结果。 我们将为联盟制定、记录和跟踪绩效指标，以便为联盟的评估提供信息 计划的成功和影响。

PROJECT SUMMARY The overarching goal of NCI's Pediatric In Vivo Testing Program (Ped-In Vivo-TP) is to improve outcomes of pediatric cancer patients and to satisfy the requirements of the Research to Accelerate Cures and Equity (RACE) for Children Act to assess the efficacy of targeted anti-cancer agents developed for adults in pediatric contexts. To coordinate the activities of the new Ped-In Vivo-TP initiative, we have assembled a team of investigators from The Jackson Laboratory (JAX) and Seven Bridges Genomics (SB) with unique combined expertise and experience with in vivo cancer models, scalable cloud-based data management and analysis systems, informatics resource development, and multi-site project coordination. Combining the complementary strengths of JAX and SB provides the ideal foundation for a coordinating center to maximize the short- and long-term impacts of the Pediatric In Vivo Testing Program for advancing the application of precision medicine in pediatric oncology. We will manage a comprehensive and cohesive testing program to advance precision medicine in pediatric oncology through effective public-private partnerships among pharmaceutical companies, regulatory agencies, funders, and research organizations. We will achieve this goal through the following aims: Aim 1: Establish and maintain the Pediatric In Vivo Testing Coordinating Center (PIVOT CC) to provide administrative and logistical support for diverse stakeholders in the Pediatric in Vivo Testing Program consortium. We will draw on our team's combined decades of experience with multi-site program management, cancer model development, standardized testing of in vivo cancer models, data management and analysis, and informatics resource development to ensure timely decision making, conformance to standard protocols, resource tracking, and effective communications within the Ped-In Vivo-TP. Aim 2: Provide data management, statistical, and bioinformatics support to ensure data security and integrity. We will leverage existing protocols and software systems at JAX and SB to identify relevant in vivo cancer models and to collect, analyze, and securely manage data generated from testing centers within the consortium. We will perform statistical and bioinformatic analyses on consortium data to reliably inform the evaluation of the efficacy of novel therapeutic agents in a pediatric oncology setting. We will develop a public- facing data portal for sharing of data and methods with the broader scientific community. Aim 3: Provide scientific coordination to maintain an efficient and effective preclinical testing pipeline. We will draw from the extensive experience of our team with in vivo pharmacology and coordination of similar consortia to manage and coordinate the testing of agents by the Ped-In Vivo-TP centers at all stages of the process, from the identification of relevant in vivo models to the generation of final technical reports and publication of results. We will develop, record, and track performance metrics for the consortium to inform the evaluation of the program's success and impact.