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

项目摘要 在了解白血病的分子基础方面的进展，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。到 为了最大限度地发挥这些发现的价值，必须证明我们有能力从分子水平上表征 个体肿瘤，并应用该信息将患者纳入治疗方案， 分子谱到特异性靶向治疗-所有这些都在有效的癌症治疗试验的背景下 系统为研究创造性和新发现的快速临床转化创造氛围 成人白血病患者的治疗方法，肿瘤临床试验联盟（以下简称 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）和布罗德研究所已选择在2014年联手，并创建了一个综合 白血病转化科学中心（ITSC-L）。ITSC-L的总体目标是确定遗传 以及表征白血病转化的分子畸变，了解它们对治疗的贡献 反应或耐药性，并利用这些信息设计合理的治疗试验， 分子畸变到新兴的靶向治疗。这些目标将通过精心 选择涉及在Alliance和SWOG内工作的杰出研究者的试点研究，以及 在团体之外工作。最终，这些努力不仅应导致 白血病患者的临床结果，但预计将产生适用于其他 癌症的类型。该方案的具体目标如下： 具体目标1。维持白血病综合转化科学中心（ITSC-L）， 促进在NCI国家临床试验网络（NCTN）内工作的研究人员的研究 通过提供和协调资金、专门知识、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例的遗传和表观遗传标记物和治疗靶点 这改变了目前对白血病患者的治疗方法，提高了他们的治愈率;具体目标3。快速 将高影响力的相关科学整合到白血病早期和晚期治疗试验中， 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.