Specialized Program of Research Excellence in Myeloma

描述（由申请人提供）：Dana Farber/哈佛癌症中心（DF/HCC）多发性骨髓瘤（MM）SPORE由5个研究项目和4个核心以及职业发展和发展研究项目组成。 它充分利用了哈佛附属机构的研究、临床专业知识和设施的互补优势，包括达纳法伯癌症研究所、哈佛医学院、哈佛公共卫生学院、马约诊所、康奈尔大学韦尔医学院和亚利桑那大学医学院。 该SPORE代表了这些机构的MM研究小组之间的合作努力，这些研究小组在基础和临床科学互动与合作方面有着长期的承诺和良好的记录。 我们在临床前细胞和分子研究以及联合临床方案方面建立了合作关系。 该小组作为一个整体长期致力于MM转化研究，并提供必要的行政、基础科学和临床基础设施。 在这些完善的中心，每年评估750多名新的MM患者，以及10，000名患有浆细胞恶液质的确诊患者的门诊就诊。 评估的疾病范围从意义不明的单克隆丙种球蛋白病到浆细胞白血病。 每个中心都有适当的科学和机构审查委员会，以及方案审核和质量控制中心，以进行尖端的转化研究。 目前有超过50个积极的协议评估治疗，包括新药，免疫治疗，改善干细胞移植，并在MM的支持疗法。这个大的合并患者基础，确保快速增长和评估的治疗效果的新药物在这个程序中确定。该项目临床前和临床部分的成功将取决于这些中心之间的协同作用和沟通。 为了确保这一目标，我们建立了一个互联网网站，允许所有主要研究者访问联合研究工作中产生的临床前数据。 同样，来自联合临床试验方案的数据也将存放在这个安全的网站上，以便在这些网站上无缝和统一地进行临床研究。目前，有系统的质量控制交换骨髓和血液样本，用于相关的基础实验室研究。 DF/HCC骨髓瘤SPORE的总体主题是识别和评估新型靶向治疗。 我们的大多数项目从一开始就源于临床研究，这一事实突出了SPORE的转化性质。 具体项目包括（1）蛋白酶体导向的新型骨髓瘤治疗;（2）针对骨髓瘤治疗的端粒扩增机制;（3）MUC 1作为多发性骨髓瘤的治疗靶点;（4）针对MM遗传缺陷的新型治疗药物;以及（5）从MGUS到骨髓瘤进化的分子标记。 核心资源包括行政和通信核心，组织核心，功能基因组学和生物信息学核心和生物统计学核心。 因此，该计划代表了在MM基础和临床研究专业知识方面具有独特和长期记录的机构的综合努力，现在联合起来，更快地将合理的新型靶向治疗从实验室转移到临床方案，以改善MM患者的结局。

DESCRIPTION (provided by applicant): The Dana Farber/Harvard Cancer Center (DF/HCC) multiple myeloma (MM) SPORE consists of 5 Research Projects and 4 Cores, as well as the Career Development and Developmental Research Programs. It capitalizes on the complementary strengths of the research, clinical expertise and facilities of the Harvard affiliated institutions including Dana Farber Cancer Institute, Harvard Medical School, Harvard School of Public Health, as well as the Mayo Clinic, the Cornell University Weil Medical College; and the University of Arizona Medical School. This SPORE represents a collaborative effort between MM research groups from these institutions with a long-standing commitment and track record of basic and clinical scientific interactions and cooperation. We have established a collaborative effort, both in preclinical cellular and molecular studies and in joint clinical protocols. The group as a whole has a long-term commitment to translational MM research, with the necessary administrative, basic science, and clinical infrastructure. At these well established centers, more than 750 new patients with MM are evaluated annually, as well as 10,000 outpatient visits for established patients with plasma cell dyscrasias. The spectrum of diseases evaluated spans from monoclonal gammopathy of unclear significance to plasma cell leukemia. Each center has appropriate scientific and institutional review boards, as well as protocol audit and quality control centers, to conduct cutting edge translational research. There are presently more than 50 active protocols evaluating therapies including novel drugs, immune treatments, improved stem cell transplantation, and supportive therapies in MM. This large combined patient base assures rapid accrual and evaluation of the therapeutic efficacy of novel agents identified in this program. Success of both the preclinical and clinical components of this Program will be dependent upon synergy and communication between these centers. To assure this end, we have set up an Internet site that allows access to all the Principal Investigators to the preclinical data generated in joint research efforts. Similarly, data from the joint clinical protocol trials will also be deposited in this secure web site to allow a seamless and uniform conduct of clinical studies at these sites. Currently there is systematic quality-controlled exchange of bone marrow and blood samples for correlative basic laboratory studies. The overall theme of the DF/HCC myeloma SPORE is to identify and evaluate novel targeted therapies. The translational nature of the SPORE is highlighted by the fact that most of our projects have emanated from clinical studies from the outset. Specific Projects are (1) Proteasome-Directed Novel Myeloma Therapies; (2) Targeting Telomere Expansion Mechanisms For Myeloma Therapy; (3) MUC1 as a Therapeutic Target in Multiple Myeloma; (4) Novel Therapeutics Targeting Genetic Abnormalities in MM; and (5) Molecular Markers of Evolution from MGUS To Myeloma. Core resources include Administrative and Communication Core, Tissue Core, Functional Genomics and Bioinformatics Core, and Biostatistics Core. This Program therefore represents the integrated efforts of institutions with a unique and long track record of basic and clinical research expertise in MM, now joining together to more rapidly move rational novel targeted therapies from the laboratory to clinical protocols to improve patient outcome in MM.