Translational Research Center to Expedite Novel Therapies in Cystic Fibrosis

转化研究中心将加快囊性纤维化的新疗法

• 批准号: 9762084
• 负责人: Bonnie W Ramsey
• 金额: $ 121.3万
• 依托单位: SEATTLE CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-07-06 至 2020-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9762084
• 关键词: Acute; Affect; Antibiotic Therapy; Area; Basic Science; Biological; Biological Assay; Biological Markers; Cell physiology; Cells; Center for Translational Science Activities; Child; Chronic lung disease; Clinical; Clinical Microbiology; Clinical Research; Clinical Trials; Clinical Trials Design; Collaborations; Communities; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA; Data; Development; Digestive System Disorders; Direct Costs; Disease; Disease Progression; Doctor of Philosophy; Drug Screening; Ecosystem; Environmental Risk Factor; Epithelial; Epithelial Cells; Fostering; Functional disorder; Funding; Gastroenterology; Gastrointestinal tract structure; Genetic Diseases; Genomics; Grant; Health; Hepatobiliary; Human; Immune; Immune response; Immunology; Infant; Infection; Inflammation; Inflammatory; Infrastructure; Inhalation; Institution; Interdisciplinary Study; International; Intestines; Islet Cell; Kidney; Laboratories; Leadership; Link; Lung; Lung infections; Malnutrition; Measures; Medicine; Metabolic; Metabolism; Methodology; Microbiology; Mutation; National Institute of Diabetes and Digestive and Kidney Diseases; Nutritional; Observational Study; Outcome; Outcome Measure; Pancreas; Pathogenesis; Patients; Pediatrics; Pharmaceutical Preparations; Physiological; Process; Proteins; Pseudomonas aeruginosa; Recording of previous events; Research; Research Institute; Research Personnel; Resources; Respiratory System; Scientist; Specimen; Testing; Tissues; Tobramycin; Translational Research; Translations; United States National Institutes of Health; Universities; Vision; Washington; base; children with cystic fibrosis; clinical database; comparative; comparative effectiveness; cystic fibrosis patients; data resource; design; drug development; effectiveness research; experience; genome sciences; gut microbiome; improved; in vivo; interest; medical schools; microbiome research; mid-career faculty; next generation; novel therapeutic intervention; novel therapeutics; nutrition; professor; programs; public health relevance; ranpirnase; repository; response; small molecule; therapeutic development

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.

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）。这个拟议的P30中心项目增强了这些机构现有的强大的基础和临床研究基础，目前的研究资金总额超过2000万美元，CF相关基础设施资金额外增加了500万美元，包括19个部门和/或分部的48名UWSOM研究人员。UWSOM在CF相关的转化研究方面有着悠久的卓越历史，成功开发了吸入妥布霉素（TOBI(r)），并在CFF支持的治疗学开发网络中处于领导地位。该P30的研究将集中在四个具体目标上：1)提供资源和专业知识，以加快有希望的新治疗方法的开发，以纠正功能失调的CFTR及其继发后果，包括消化不良和营养不良、肝胆疾病和慢性肺病。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资源库来加强转化研究。利用这些共享的标本和数据资源来开发改进的分析、生物标志物和临床结果测量。3)加强对囊性纤维化患者胃肠道和呼吸道细菌生态系统的进化和由此产生的宿主反应以及它们如何相互作用的理解