Gene- and Cell-based therapies for LVAD patients

针对 LVAD 患者的基因和细胞疗法

• 批准号: 7878703
• 负责人: DAVID ANDREW BULL
• 金额: $ 35.13万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-09-30 至 2011-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7878703
• 关键词: Academic Medical Centers; Arizona; Bone Marrow; Cardiac; Cardiology; Cardiomyopathies; Cell Therapy; Cells; Cities; Clinical; Clinical Research; Clinical Trials; Colorado; Community Practice; Congestive Heart Failure; Coordination and Collaboration; Destinations; Enrollment; Genes; Health Sciences; Healthcare Systems; Heart Transplantation; Heart failure; Hospitals; Idaho; Image; Immunosuppressive Agents; Injection of therapeutic agent; Ischemia; Laboratories; Left; Mechanics; Medical center; Medicine; Mononuclear; Montana; Myocardial; Natural regeneration; Nevada; Operative Surgical Procedures; Patients; Pharmaceutical Preparations; Phase; Phase I Clinical Trials; Plant Roots; Polymers; Principal Investigator; Program Development; Protocols documentation; Recording of previous events; Recovery; Recruitment Activity; Research Personnel; Safety; Services; Sodium Chloride; Staging; Stem cells; Transplantation; United States; United States Department of Veterans Affairs; Universities; Utah; VEGFA gene; Vascular Endothelial Growth Factors; Veterans; Wyoming; abstracting; angiogenesis; base; expression vector; gene delivery system; gene therapy; novel; patient population; poly(ethylenimine)-co-(N-(2-aminoethyl) ethyleneimin)-co-N-(N-cholesteryloxycarbonyl-(2-aminoethyl)ethylenimine); programs; repaired; safety study; skills; stem cell population; ventricular assist device

DESCRIPTION (provided by applicant): SINCE 1985, the cardiology and cardiothoracic departments of the three major tertiary medical centers (University of Utah Hospitals, LDS Hospital, and Salt Lake City Veterans Affair Medical Center) have integrated into a single multihospital, multidepartment program in heart failure/cardiac transplant that serves a broad geographical region of the Western United States. This program, the Utah Transplantation Affiliated Hospitals (UTAH Network), services patients in Utah, Montana, Wyoming, Idaho, Nevada, Northern Arizona and Western Colorado. The UTAH network was the leading enroller for the REMATCH trial, a pivotal trial that established the efficacy of left ventricular assist devices (LVAD) as a destination therapy. Thus, the UTAH network is a clinical network with an extensive heart failure patient base, deep roots in the regional practice community, a long tradition of collaboration and coordination, and a proven history of recruiting patients for national heart failure trials. The UTAH investigators propose to treat LVAD patients with gene- and cell-based therapies that will induce angiogenesis, cardiac regeneration and repair, and enhance cardiac function. The LVAD patient population offers: 1) the unique opportunity to intensively study the functional and histopathological effects of these experimental medicines; 2) margin of safety for clinical trials involving novel gene- and cell-based therapies; 3) rapidly growing numbers of patients available for enrollment. Specifically the UTAH network proposes to treat heart failure patients undergoing LVAD placement with: Project 1- an ischemia-inducible VEGF expression vector using non-viral polymer based gene therapy. Project 2- direct injections of specific stem cell populations. The use of gene- and cell-based therapies at the University of Utah is supported by: 1) General Clinical Research Center with a 40 year old history; 2) Pioneering program developing non-viral polymer gene delivery systems 3) GMP clinical stem cell laboratory approved to support phase I and II protocols 4) Advance imaging core with expertise in LVAD and heart failure patients 5) Clinical Research Skills Development Program. (End of Abstract)

描述（由申请人提供）： 自1985年起，三大三甲中心心内科、心胸科 （犹他大学医院、摩门教医院和盐湖城退伍军人事务医疗中心） 整合到一个单一的多医院、多科室的心力衰竭/心脏移植项目中，该项目服务于美国西部广大的地理区域。该计划称为犹他州移植附属医院（犹他州网络），为犹他州、蒙大拿州、怀俄明州、爱达荷州、内华达州、亚利桑那州北部和科罗拉多州西部的患者提供服务。犹他州网络是 REMATCH 试验的主要招募者，该试验确立了左心室辅助装置 (LVAD) 作为目标治疗的功效。因此，犹他州网络是一个临床网络，拥有广泛的心力衰竭患者基础、在区域实践社区中扎根深厚、合作与协调的悠久传统以及招募患者参加国家心力衰竭试验的良好历史。 犹他州研究人员建议用基于基因和细胞的疗法治疗 LVAD 患者，这些疗法将诱导血管生成、心脏再生和修复，并增强心脏功能。 LVAD 患者群体提供了：1）深入研究这些实验药物的功能和组织病理学影响的独特机会； 2) 涉及新型基因和细胞疗法的临床试验的安全边际； 3）可供登记的患者数量迅速增加。具体来说，UTAH 网络建议通过以下方法治疗接受 LVAD 放置的心力衰竭患者： 项目 1——使用基于非病毒聚合物的基因疗法的缺血诱导型 VEGF 表达载体。 项目2-直接注射特定干细胞群。 犹他大学使用基于基因和细胞的疗法得到了以下机构的支持： 1) 拥有 40 年历史的综合临床研究中心； 2) 开发非病毒聚合物基因传递系统的开创性计划 3) GMP 临床干细胞实验室获批支持 I 期和 II 期方案 4) 拥有 LVAD 和心力衰竭患者专业知识的先进成像核心 5) 临床研究技能发展计划。 （摘要完）