Gene- and Cell-based therapies for LVAD patients

描述（由申请人提供）： 自1985年以来，三大三级医疗中心的心脏科和心胸科 （犹他州大学医院、LDS医院和湖城退伍军人事务医疗中心） 整合到一个单一的多医院，多部门的心力衰竭/心脏移植计划，服务于美国西部的广泛地理区域。这个项目，即犹他州移植附属医院（UTAH网络），为犹他州、蒙大拿州、怀俄明州、爱达荷州、内华达州、北方亚利桑那州和西部科罗拉多的患者提供服务。犹他州网络是REMATCH试验的主要招募者，该试验是一项关键性试验，旨在确定左心室辅助装置（LVAD）作为目标治疗的有效性。因此，犹他州网络是一个临床网络，具有广泛的心力衰竭患者基础，在区域实践社区中有着深厚的根基，具有悠久的合作和协调传统，以及招募患者进行国家心力衰竭试验的历史。 犹他州大学的研究人员建议用基因和细胞为基础的疗法来治疗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患者人群提供了：1）深入研究这些实验性药物的功能和组织病理学作用的独特机会; 2）涉及新型基因和细胞治疗的临床试验的安全范围; 3）可供招募的患者数量迅速增长。具体而言，犹他州网络建议用以下方法治疗接受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治疗的缺血诱导型VEGF表达载体。 项目2-直接注射特定的干细胞群。 犹他州大学基于基因和细胞疗法的使用得到以下方面的支持：1）具有40年历史的综合临床研究中心; 2）开发非病毒聚合物基因递送系统的开创性计划3）GMP临床干细胞实验室获批支持I期和II期方案4）先进的成像核心，具有LVAD和心力衰竭患者的专业知识5）临床研究技能发展计划。(End摘要）

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)