COG NCTN Integrated Translational Science Center for Hematopoietic Malignancies in Children

项目摘要 包括白血病和淋巴瘤在内的血液系统恶性肿瘤占所有癌症的近一半， 是导致儿童疾病死亡的主要原因。儿童肿瘤学小组(COG)带头 努力改善癌症儿童的预后，尽管在过去的几年中存活率有了显著的提高 几十年来，复发仍然是一个主要问题，幸存者可能会遭受长期的并发症 细胞毒疗法。更好地了解恶性肿瘤的生物学有助于更理性、更有针对性地 治疗方法，将非选择性细胞毒治疗的需要或强度降至最低。是这样的 有针对性的方法已经将一些疾病从一致致命转变为高度可治愈(例如，CML， APL(APL：行情)，但缺乏可操作的目标阻碍了针对性干预在 恶性血液病。作为其重点关注的一部分，优化儿童癌症的存活率和改善 为了提高癌症幸存者的生活质量，COG通过与NCI的合作开展了许多 发现阶段研究，以确定儿童癌症的生物学基础，目的是确定特定的 在其临床试验中为更适当的风险分层和定向治疗制定目标。随着新兴数据的出现 在儿科(和成人)发现阶段的研究中，COG准备采取下一步行动，确定可操作的 治疗干预的靶点。作为这些努力的一部分，COG创建了网络集团集成 旨在为治疗驱动的翻译提供资源的翻译科学中心(ITSC) 研究。COG造血ITSC(HM-ITSC)已建立起智力(领导力)和体力 资源(资源实验室、生物群落等)确保快速确定可采取行动的目标 通过精心设计的翻译研究项目，并通过 精心构建的机制应用于临床。HM-ITSC已构建并集成到功能 COG的运作，与COG行动办公室、疾病委员会、网络小组有直接联系 数据和统计中心和发展治疗，以促进可采取行动的快速流动 在NCTN临床试验中将生物学数据转化为临床实施。恶性血液病 (HM)是儿童癌症的主要原因，需要新的治疗靶点来优化结果。 COG HM-整合到COG职能组织的综合翻译科学中心，将 提供资源支持儿童白血病和淋巴瘤的翻译研究，以 确定具有临床意义的靶点并将其纳入临床试验。

Project Summary Hematopoietic malignancies including leukemias and lymphomas contribute to nearly half of all cancers and are a major cause of death from disease in children. The Children’s Oncology Group (COG) has spearheaded efforts to improve outcome in children with cancer, and despite dramatic improvements in survival over the last several decades, relapse remains a major problem, and survivors may suffer long term complications from the cytotoxic therapy. Better understanding of the biology of malignancies allows for more rational, targeted approaches to therapy, minimizing the need or the intensity of the non-selective cytotoxic therapies. Such targeted approaches have transformed some diseases from uniformly fatal to highly curable ones (e.g., CML, APL), but a paucity of actionable targets has prevented broader application of targeted interventions in hematopietic malignancies. As part of its focus on optimizing the survival in pediatric cancers and improving quality of life for the cancer survivors, COG through partnerships with NCI has conducted a number of discovery phase studies to identify the biologic basis of cancer in children, with the goal of identifying specific targets for more appropriate risk stratification and directed therapies in its clinical trials. With the emerging data from pediatric (and adult) discovery phase studies, COG is poised to take the next step in identifying actionable targets for therapeutic intervention. As part of these efforts, COG has created the Network Group Integrated Translational Science Centers (ITSCs) aimed at providing resources for therapeutically driven translational research. COG Hematopoietic ITSC (HM-ITSC) has put into place intellectual (leadership) and physical resources (resource laboratories, biospecimens, etc.) to ensure rapid identification of actionable targets through carefully designed translational research projects, and fostering the experimental data through a carefully structured mechanism to clinical application. HM-ITSC is structured and integrated into the functional operations of the COG, with direct links to the COG Operations Office, Disease Committees, Network group Data and Statistics Center and Developmental Therapeutics in order to facilitate rapid flow of actionable translational biology data to clinical implementation within NCTN clinical trials. Hematopoietic Malignancies (HM) are a major cause of cancer in children and novel therapeutic targets are needed to optimize outcome. COG HM-Integrated Translational Science Center, integrated into the functional organization of COG, will provide resources to support translational research in pediatric leukemias and lymphomas towards identification of clinically meaningful targets and their incorporation into clinical trials.