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

描述(由申请人提供)：在了解白血病分子基础方面的进展，以及不断涌现的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，必须证明我们有能力对单个肿瘤进行分子特征描述，并利用这些信息将患者纳入治疗方案，使不同的分子特征与特定的靶向治疗相匹配--所有这些都是在有效的癌症治疗试验系统的背景下进行的。为了营造一种既有研究创造力又能迅速将新发现转化为成人白血病患者的治疗方法的氛围，肿瘤学临床试验联盟(以下简称联盟)白血病相关科学委员会、上海奥克兰大学白血病转化医学小组委员会和布罗德研究所选择联合成立白血病综合转化科学中心(L)。ITSC-L的总体目标是识别表征白血病转化的遗传和分子异常，了解它们对治疗反应或耐药的贡献，并利用这些信息设计合理的治疗试验，使特定的分子异常与新出现的匹配。 有针对性的治疗。这些目标将通过仔细挑选试点研究和合作项目来实现，这些项目包括在联盟和SWOG内部工作的杰出调查人员以及在这两个组织之外工作的人员。最终，这些努力不仅应该导致白血病患者临床结果的显著改善，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，通过为具有临床意义的创新研究提供和协调资金、专业知识、技术、组织和数据，促进在国家临床试验网络系统内和系统外工作的研究人员的研究；具体目标2.确定新的预测和预测遗传和表观遗传学标记和治疗靶点，以开发创新的治疗范例，改变目前对白血病患者的治疗方法，并提高他们的治愈率；具体目标3.将高影响力的相关科学迅速整合到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.