MPD Research Consortium

MPD研究联盟

• 批准号: 8924960
• 负责人: Ronald Hoffman
• 金额: $ 648.09万
• 依托单位: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-07-01 至 2017-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8924960
• 关键词: Affect; Alleles; Animal Model; Basic Science; Biological Markers; Biometry; Cells; Characteristics; Chimeric Proteins; Chronic Myeloid Leukemia; Clinical; Clinical Research; Clinical Treatment; Clinical Trials; Clinical Trials Design; Collection; Data; Databases; Development; Discipline; Disease; Drug Targeting; Effectiveness; Evaluation; Exons; Foundations; Genes; Genetic; Geographic Locations; Goals; Hematologic Neoplasms; Hematopoietic stem cells; Hemorrhagic Thrombocythemia; Individual; Institution; Instruction; International; Janus kinase 2; Knowledge; Laboratories; Lead; Link; Marrow; Modeling; Molecular Target; Mus; Myelofibrosis; Myeloproliferative disease; Natural History; Normal Cell; Online Systems; Outcome; Pathogenesis; Pathology; Patients; Pharmaceutical Preparations; Phase; Philadelphia Chromosome; Phosphotransferases; Play; Point Mutation; Polycythemia Vera; Primary Myelofibrosis; Research; Research Infrastructure; Research Personnel; Risk Reduction; Role; Scientist; Single Nucleotide Polymorphism; Somatic Mutation; Specificity; Specimen; Splenomegaly; Stem cells; Symptoms; Therapeutic; Tissue Banking; Tissue Banks; Tissue Sample; Tyrosine Kinase Inhibitor; United States; base; bcr-abl Fusion Proteins; cellular targeting; data management; design; drug development; effective therapy; improved; inhibitor/antagonist; meetings; member; mouse model; novel; novel therapeutics; overexpression; programs; response; sample collection; skills; small molecule; success; therapeutic target; therapy design; trafficking; treatment program; treatment strategy; web site

DESCRIPTION (provided by applicant): The Myeloproliferative Disorders Research Consortium (MPD-RC) focuses on the Philadelphia chromosome negative myeloproliferative neoplasms (MPN) including polycythemia vera (PV), essential thrombocythemia (ET) and primary myelofibrosis (PMF) and has the following goals 1) Establish a multi-institutional international research group which will coordinate and facilitate basic and clinical research dealing with the cellular and genetic basis of the MPN. 2) Establish a multi-institutional Clinical Consortium to enable uniform, high volume sample collection, storage and distribution. 3) Perform rationally designed clinical trials in patients with MPN at multiple institutions. 4) Maintain an interactive website for MPD Consortium investigators, a sophisticated international tissue bank and an on-line database that will allow for integration of basic and clinical research. This unique interactive relationship between talented basic researchers and clinical scientists will permit the MPD-RC to develop novel clinical treatment programs for MPN and to identify specific biomarkers that will be useful as indicators of therapeutic response and/or risk reduction. This program has six major projects: Project 1: Genetic Basis of Polycythemia Vera, Project Leader: J. T. Prchal; Project 2: A Novel Murine Model for MPN: Pathology and Therapy of NF-E2 Overexpression, Project Leader: H. L. Pahl; Project 3: Animal Models of Polycythemia Vera, Project Leader: J. L. Spivak; Project 4: Mouse Models of Myelofibrosis, Project Leader: A. Migliaccio; Project 5: Abnormal Stem Cell Trafficking in Myelofibrosis, Project Leader: R. Hoffman; Project 6: MPD Clinical Consortium which will pursue clinical trials in PV. ET and PMF, Project Leader: Lewis Silverman. The 6 projects will be supported by 3 cores: Core A, Administrative Core, PI: R. Hoffman; Core B: Biostatistics and Data Management; PI: J. A. Goldberg and Co-PI R. Marchioli; Core C; Tissue Bank, PI: R. Weinberg. These unique interactions between clinical and laboratory investigators which are interwoven within the MPD-RC will ultimately result in improved understanding of the pathobiology of the MPN as provide well improved strategies that will assist in the management of MPN patients.

描述（由申请人提供）：脊髓增生性疾病研究联盟（MPD-RC）着重于费城染色体负阴性骨髓增生性肿瘤（MPN）（MPN），包括多余的肿瘤（MPN），包括多余的肿瘤（PV）（PV），包括基本的血小板（ESTROMIA（ET）和一级骨骼骨骼（PMF），并建立了以下是一项综合目标1）并促进有关MPN细胞和遗传基础的基本和临床研究。 2）建立一个多机构的临床财团，以实现统一，大量样本收集，存储和分布。 3）在多个机构的MPN患者中进行合理设计的临床试验。 4）维护一个用于MPD财团调查员的交互式网站，一个精致的国际组织库以及一个在线数据库，该数据库将允许基础和临床研究的整合。才华横溢的基础研究人员和临床科学家之间的这种独特的互动关系将允许MPD-RC为MPN制定新颖的临床治疗计划，并确定特定的生物标志物，这些标志物将作为治疗反应和/或降低风险的指标有用。该计划有六个主要项目：项目1：Polycythemia Vera的遗传基础，项目负责人：J。T. Prchal；项目2：MPN的新型鼠模型：NF-E2过表达的病理和治疗，项目负责人：H。L. Pahl；项目3：Vera的动物模型，项目负责人：J。L. Spivak；项目4：骨髓纤维化的小鼠模型，项目负责人：A。Migliaccio；项目5：项目负责人：R。Hoffman；项目6：将在PV中进行临床试验的MPD临床联盟。 ET和PMF，项目负责人：刘易斯·西尔弗曼（Lewis Silverman）。这6个项目将得到3个核心的支持：核心A，行政核心，PI：R。Hoffman；核心B：生物统计学和数据管理； PI：J。A. Goldberg和Co-Pi R. Marchioli；核心C; PI纸币：R。Weinberg。在MPD-RC中交织在一起的临床和实验室研究人员之间的这些独特的相互作用最终将改善对MPN病理生物学的了解，因为提供了很好的改进策略，这些策略将有助于管理MPN患者。