ITSC for Leukemia: Novel Molecular strategies for NCTN "Individualized" Therapies

项目概要 对白血病分子基础的理解取得进展，以及新兴创新的财富 药物化合物，为改善患者的临床结果带来了巨大的机会。至 为了最大限度地发挥这些发现的价值，必须证明我们的分子表征能力 个体肿瘤，并应用此信息将患者纳入与不同肿瘤相匹配的治疗方案 从分子特征到特定靶向治疗——所有这些都在有效的癌症治疗试验的背景下进行。 系统。为研究创造力和新发现的快速临床转化创造氛围 肿瘤学临床试验联盟（以下简称“联盟”）对成人白血病患者的治疗方法进行了研究 联盟）白血病相关科学委员会 (A-LCSC) 和 SWOG 白血病转化医学 小组委员会 (S-LTMS) 和布罗德研究所选择于 2014 年联手，创建了一个综合 白血病转化科学中心 (ITSC-L)。 ITSC-L 的总体目标是识别遗传 和表征白血病转化的分子畸变，了解它们对治疗的贡献 反应或抵抗，并利用这些信息来设计符合特定情况的合理治疗试验 新兴靶向治疗的分子畸变。这些目标将通过精心设计来实现 选择试点研究，其中涉及联盟和 SWOG 内工作的杰出研究人员以及那些 在团体之外工作。最终，这些努力不仅应该导致显着改善 白血病患者的临床结果，但预计会产生适用于其他疾病的前沿发现 癌症的类型。该计划的具体目标如下： 具体目标 1. 维持一个白血病综合转化科学中心 (ITSC-L)，该中心将 促进 NCI 国家临床试验网络 (NCTN) 内研究人员的研究 通过提供和协调资金、专业知识、技术、组织和 具有临床相关性的创新研究数据；具体目标 2. 确定新的预后和预测 用于开发创新治疗范式的遗传和表观遗传标记以及治疗靶点 改变目前治疗白血病患者的方法并提高他们的治愈率；具体目标 3. 迅速 将高影响力的相关科学整合到白血病的早期和晚期治疗试验中，并得到以下支持 NCTN。

PROJECT SUMMARY Advances in understanding the molecular basis of leukemia, together with the wealth of emerging innovative pharmaceutical compounds, have brought great opportunities to improve the clinical outcome of patients. To maximize the value of these discoveries, it is imperative to demonstrate our ability to molecularly characterize individual tumors and apply this information to enroll patients onto therapeutic protocols that match distinct molecular profiles to specific targeting treatments—all within the context of an effective cancer therapeutic trials' system. To create an atmosphere for both research creativity and rapid clinical translation of novel discoveries into treatment approaches of adult leukemia patients, the Alliance for Clinical Trials in Oncology (hereafter called Alliance) Leukemia Correlative Science Committee (A-LCSC), and the SWOG Leukemia Translational Medicine Subcommittee (S-LTMS) and the Broad Institute have elected to joined forces in 2014 and created an Integrated Translational Science Center for Leukemia (ITSC-L). The overall goal of the ITSC-L is to identify the genetic and molecular aberrations that characterize leukemic transformation, understand their contribution to therapeutic response or resistance, and utilize this information for the design of rational therapeutic trials that match specific molecular aberrations to emerging targeted therapies. These goals will be achieved through the carefully selection of Pilot Studies that involve outstanding investigators working within Alliance and SWOG and those working outside the groups. Ultimately, these efforts should not only result in a significant improvement in the clinical outcome for leukemia patients, but are expected to generate cutting-edge findings applicable to other types of cancer. The Specific Aims of this Program are the following: Specific Aim 1. To maintain an Integrated Translational Science Center for Leukemia (ITSC-L) that will promote research among investigators working within the NCI National Clinical Trials Network (NCTN) system and outside the system by providing and coordinating funding, expertise, technology, tissue and data for innovative studies with clinical relevance; Specific Aim 2. To identify novel prognostic and predictive genetic and epigenetic markers and therapeutic targets for the development of innovative therapeutic paradigms that transform the current approach to leukemia patients and improve their cure rate; Specific Aim 3. To rapidly integrate high impact correlative science into early and late phase therapeutic trials of leukemia supported by the NCTN.