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

ITSC​​ 治疗白血病：NCTN 的新型分子策略：“个体化”治疗

• 批准号: 9256443
• 负责人: CLARA D BLOOMFIELD
• 金额: $ 64.39万
• 依托单位: OHIO STATE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-05-02 至 2019-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9256443
• 关键词: Adopted; Adult; Area; Biological Markers; Cancer Biology; Center for Translational Science Activities; Clinical; Clinical Trials; Creativeness; Data; Development; Disease; Enrollment; Funding; Genetic; Genetic Markers; Goals; Individual; Institutes; International; Malignant Neoplasms; Molecular; Molecular Abnormality; Molecular Profiling; National Cancer Institute; National Clinical Trials Network; Outcome; Patient-Focused Outcomes; Patients; Pharmacologic Substance; Phase; Pilot Projects; Prognostic Marker; Protocols documentation; Reproduction spores; Research; Research Personnel; Science; Southwest Oncology Group; Stratification; System; Technology; Therapeutic; Therapeutic Trials; Tissues; United States; Work; adult leukemia; cancer clinical trial; cancer type; clinical translation; clinically relevant; design; epigenetic marker; genetic predictors; high throughput technology; improved; individualized medicine; innovation; leukemia; molecular diagnostics; molecular subtypes; new technology; novel; oncology; planetary Atmosphere; programs; public health relevance; targeted treatment; therapeutic target; therapy resistant; translational medicine; treatment response; tumor

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.

描述（由申请人提供）：对白血病分子基础的了解的进展，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个体肿瘤进行分子表征，并应用这些信息来将患者纳入治疗方案，将不同的分子特征与特定的靶向治疗相匹配——所有这些都在有效的癌症治疗试验的背景下进行' 系统。为了营造一种既能促进研究创造力，又能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（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) 和布罗德研究所已选择联手创建白血病综合转化科学中心 (ITSC-L)。 ITSC​​-L 的总体目标是识别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，使特定的分子畸变与新兴的相匹配。 靶向治疗。这些目标将通过精心挑选试点研究和合作项目来实现，这些项目涉及联盟和 SWOG 内部以及团体外部工作的杰出研究人员。最终，这些努力不仅应该显着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前沿发现。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)，促进 NCI 国家临床试验网络 (NCTN) 系统内和系统外研究人员的研究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；具体目标 2. 确定新的预后和预测性遗传和表观遗传标记以及治疗靶点，以开发创新的治疗范式，从而改变目前治疗白血病患者的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。