Translational Research Center to Expedite Novel Therapies in Cystic Fibrosis

描述（由申请人提供）：该P30囊性纤维化（CF）研究和转化核心中心（CFRTC）位于华盛顿大学医学院（UWSOM）及其附属机构西雅图儿童研究所（SCRI）。这项拟议的P30中心计划增强了这些机构现有的强大的基础和临床研究基础，目前的研究资金直接成本超过2000万美元，CF相关基础设施资金增加了500万美元，包括19个部门和/或部门的48名UWSOM研究人员。UWSOM在CF相关转化研究方面有着悠久的卓越历史，吸入性妥布霉素（TOBI（r））的成功开发和CFF支持的治疗药物开发网络的领导地位证明了这一点。该P30的研究将集中在四个具体目标：1）提供资源和专业知识，以加快开发有前途的新治疗方法，以纠正功能失调的CFTR及其继发性后果，包括消化不良和营养不良，肝胆疾病和慢性肺部疾病。2)促进P-30生物医学核心在国内和国际上的合作，通过共享与临床数据库相关的人类和细菌储存库来加强转化研究。利用这些共享的样本和数据资源开发改进的检测方法、生物标志物和临床结局指标。3)加强对囊性纤维化患者胃肠道和呼吸道中不断变化的细菌生态系统和由此产生的宿主反应以及这些相互作用的理解 影响健康。4)更好地了解CFTR功能障碍的代谢和炎症后果。该中心将由共同PD的，邦妮拉姆齐，医学博士和E。Peter Greenberg博士，CF研究人员在各自的临床研究和细菌发病机制领域都得到国际认可。他们将得到其他关键领导的支持：医学，微生物学，基因组科学和免疫学教授Samuel米勒MD，比较医学系副教授Charles Frevert，DVM，ScD，医学和儿科教授Christopher Goss MD，MPH和医学教授Pradeep Singh MD。该中心将包括一个行政核心（Ramsey和Greenberg，董事）和四个生物医学核心，微生物学（Greenberg，董事），基因组学（米勒，董事），主机响应（Frevert，董事）和临床翻译（Christopher Goss和Ramsey，董事）。核心中心将运营由Pradeep Singh博士领导的试点和可行性（P&F）计划。三个新批准的P&F项目包括在通过竞争过程选择并由内部咨询委员会批准的当前提案中。在资助期间，核心中心将专注于开发几种新的治疗方法，以改善CF患者的整体健康状况。

DESCRIPTION (provided by applicant): This P30 Cystic Fibrosis (CF) Research and Translational Core Center (CFRTC) is located at the University of Washington School of Medicine (UWSOM) and its affiliated institution, Seattle Children's Research Institute (SCRI). This proposed P30 center program enhances an existing robust basic and clinical research base at these institutions totally over $20 million direct costs in current research funding and an additional $5 million in CF related infrastructure funding, and includes 48 UWSOM investigators in 19 departments and/or divisions. The UWSOM has a long history of excellence in CF related translational research as evidenced by the successful development of inhaled tobramycin (TOBI(r)) and the leadership in the CFF supported Therapeutics Development Network. The research for this P30 will focus on four Specific Aims: 1) Provide resources and expertise to expedite development of promising new therapeutic approaches to correct dysfunctional CFTR and its secondary consequences including maldigestion and malnutrition, hepatobiliary disease and chronic lung disease. 2) Foster collaboration across P-30 biomedical cores, nationally and internationally to enhance translational research through sharing of human and bacterial repositories linked to clinical databases. Utilize these shared specimen and data resources to develop improved assays, biomarkers and clinical outcome measures. 3) Enhance understanding of the evolving bacterial ecosystems and resultant host response in the gastrointestinal and respiratory tracts of patients with cystic fibrosis and how these interactions impact health. 4) Better understand the metabolic and inflammatory consequences of CFTR dysfunction. The center will be led by Co-PD's, Bonnie Ramsey, MD and E. Peter Greenberg PhD, CF investigators both internationally recognized in their respective areas of clinical research and bacterial pathogenesis. They will be supported by other key leadership: Samuel Miller MD, Professor of Medicine, Microbiology, Genome Sciences, and Immunology, Charles Frevert, DVM, ScD, Associate Professor in the Department of Comparative Medicine, Christopher Goss MD, MPH, Professor of Medicine and Pediatrics, and Pradeep Singh MD, Professor of Medicine. The center will consist of an Administrative Core (Ramsey and Greenberg, Directors) and four biomedical cores, Microbiology (Greenberg, Director), Genomics (Miller, Director), Host Response (Frevert, Director) and Clinical Translational (Christopher Goss and Ramsey, Directors). The core centers will operate a Pilot and Feasibility (P&F) program led by Dr. Pradeep Singh. Three newly approved P&F projects are included in the current proposal chosen through a competitive process and approved by the Internal Advisory Board. During the funding period, the core center will focus on development of several novel therapeutic approaches to improve overall health for patients with CF.