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步，以及新兴创新药物化合物的丰富，为改善患者的临床结果带来了巨大的机会。为了最大限度地发挥这些发现的价值，我们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患者的治疗方案，这些治疗方案将不同的分子特征与特定的靶向治疗相匹配——所有这些都在一个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力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，肿瘤临床试验联盟（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Broad研究所决定联手创建白血病综合转化科学中心（ITSC-L）。ITSC-L的总体目标是确定表征白血病转化的遗传和分子畸变，了解它们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，以匹配特定分子畸变的出现

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.