Translational Research in Systemic Vasculitis

描述(申请人提供)：系统性血管炎是一组炎症性血管疾病。由于对这些疾病的病理生理学知识仍不完全，他们的治疗是经验性的、有毒的，在诱导持久缓解方面往往不成功。约翰·霍普金斯大学脉管炎中心主任约翰·斯通博士描述的这一转化研究项目的见解可能会导致评估脉管炎患者的新方法。斯通博士为对转化性研究感兴趣的初级研究人员提供的指导计划提供了一个大型数据库，其中包括具有良好特征的脉管炎患者、用于临床研究培训的杰出机构资源、尽职尽责的指导、约翰·霍普金斯大学内外良好的合作关系，以及对职业发展至关重要的研究基础设施。作为这项申请的计划研究目标，斯通博士将利用现有的资金和现有的合作来发展他在蛋白质组学方面的工作。他在这项工作中的合作研究员是国家癌症研究所/食品和药物管理局(NCI/FDA)临床蛋白质组学项目的合作者Emanuel Petricoin博士和Lance Liotta博士。资金来自马里兰关节炎研究联盟(Marrc)和食品和药物管理局孤儿产品办公室 发展将支持这些研究。这项工作最初的疾病焦点将是韦格纳肉芽肿，使用在韦格纳肉芽肿病Etanercept试验中收集的血清(一项由NIAMS资助的多中心试验，斯通博士是首席研究员)。在脉管炎中心，年轻研究人员可能选择关注的其他研究领域包括：1)新的治疗方法的调查；2)微生物病原体在脉管炎病因中的作用；以及3)系统性脉管炎的分子机制(在与同事的共同指导下，他们的专业知识与斯通博士的专业知识互补)，使用来自脉管炎中心的患者样本和临床数据。

DESCRIPTION (provided by applicant): Systemic vasculitis is a group of inflammatory disorders of blood vessels. Because knowledge of the pathophysiology of these conditions remains incomplete, their treatments are empiric, toxic, and often unsuccessful in inducing durable remissions. Insights derived from this translational research program described by Dr. John Stone, Director of the Johns Hopkins Vasculitis Center, may lead to novel methods of evaluating vasculitis patients. Dr. Stone's mentoring program for junior investigators interested in translational research provides access to a large database of well-characterized patients with vasculitis, outstanding institutional resources for clinical research training, committed mentoring, well-established collaborations within and outside of Johns Hopkins, and a research infrastructure that is essential to career development. As a programmatic research goal in the context of this application, Dr. Stone will employ current funding and existing collaborations to develop his work in proteomics. His co-investigators in this effort are collaborators at the National Cancer Institute/Food & Drug Administration (NCI/FDA) Clinical Proteomics Program, Dr. Emanuel Petricoin and Dr. Lance Liotta. Funding from the Maryland Arthritis Research Consortium (MARRC) and from the Food & Drug Administration's Office of Orphan Products Development will support these studies. The initial disease focus of this work will be Wegener's granulomatosis, using sera collected in the Wegener's Granulomatosis Etanercept Trial (A multicenter, NIAMS-funded trial in which Dr. Stone is the Principal Investigator). Other areas of research at the Vasculitis Center on which young investigators may choose to focus include: 1) the investigation of novel therapeutic approaches; 2) the role of microbial pathogens in the etiology of vasculitis; and, 3) molecular mechanisms of systemic vasculitis (under co-mentoring arrangements with colleagues whose expertise complements that of Dr. Stone), using patient samples and clinical data from the Vasculitis Center.