ITSC for Leukemia: Novel Molecular Strategies for NCTN: "Individualized" Therapie

描述（由申请人提供）：对白血病分子基础的理解的进展，以及大量新兴的创新药物化合物，为改善患者的临床结局带来了巨大的机会。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对单个肿瘤进行分子表征，并将这些信息应用于将不同分子特征与特定靶向治疗相匹配的治疗方案中，所有这些都在有效的癌症治疗试验系统的背景下进行。为了创造一种研究创造性和将新发现快速临床转化为成人白血病患者治疗方法的氛围，肿瘤学临床试验联盟（Alliance for Clinical Trials in Oncology）（以下简称联盟）白血病相关科学委员会（A-LCSC），SWOG白血病转化医学小组委员会（S-LTMS）和布罗德研究所选择联合起来，创建一个白血病综合转化科学中心（ITSC-L）。ITSC-L的总体目标是确定白血病转化的遗传和分子畸变特征，了解其对治疗反应或耐药性的贡献，并利用这些信息设计合理的治疗试验，将特定的分子畸变与新出现的白血病转化相匹配。 靶向治疗。这些目标将通过仔细选择试点研究和合作项目来实现，这些项目涉及在联盟和SWOG内部工作的杰出研究人员以及在这些小组之外工作的研究人员。最终，这些努力不仅会显著改善白血病患者的临床结果，而且有望产生适用于其他类型癌症的尖端发现。该计划的具体目标如下：具体目标1。创建白血病综合转化科学中心（ITSC-L），通过为具有临床相关性的创新研究提供和协调资金、专业知识、技术、组织和数据，促进在NCI国家临床试验网络（NCTN）系统内和系统外工作的研究人员的研究;具体目标2。确定新的预后和预测性遗传和表观遗传标记物和治疗靶点，以开发创新的治疗模式，改变目前对白血病患者的治疗方法并提高其治愈率;具体目标3。将高影响力的相关科学快速整合到NCTN支持的白血病早期和晚期治疗试验中。

DESCRIPTION (provided by applicant): 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 characterizing 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), the SWOG Leukemia Translational Medicine Subcommittee (S-LTMS) and the Broad Institute have elected to join forces and create 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 and Collaborative Projects 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 create 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 o leukemia supported by the NCTN.