Symposium on Emerging Cardiac Cellular Therapies

描述（由申请人提供）： 该补助金是为了支持R-13申请一个非经常性的为期一天的会议，题为：新兴的心脏细胞疗法：将cGMP联邦法规纳入临床试验。研讨会将于2005年9月8日星期四（下午）和9月9日星期五（上午）在马里兰州巴尔的摩的喜来登内港酒店举行。本次研讨会旨在将CBER的心脏病学家、造血干细胞移植者、研究人员和FDA监管人员聚集在一起，讨论用于治疗ST段抬高MI的心脏细胞疗法中的以下细胞类型的作用：骨髓单核细胞;动员的外周血干细胞;富集的细胞亚群（即，CD 34+或CD 133细胞）;成肌细胞;自体心脏干细胞;脂肪组织干细胞;间充质干细胞。这些心脏细胞治疗临床试验必须根据与细胞治疗处理（cGMP）相关的新FDA法规进行。为了确保研究者的依从性，患者的安全性和保护公众健康，涉及使用细胞治疗最终产品的研究提出了关于当前和未来临床试验的这些问题。使用每种细胞类型背后的最新科学数据是什么？每种细胞类型的临床结果如何？每种细胞类型都涉及什么样的加工过程？是否有合适的动物模型？在心脏损伤的整体治疗中，每种细胞类型的“宣传”功能是什么？每种细胞类型的适当管理框架是什么？需要解决的生物制品/器械“组合产品”问题是什么？目标是：目标1：收集心脏细胞治疗临床试验的最新数据;目标2：确定各种心脏细胞治疗方法所涉及的实验室过程;目标3：确定心脏修复临床细胞治疗研究的关键障碍，并确定围绕这些障碍的途径;目标4：让心脏细胞治疗研究者参与关于其试验监管框架的对话;目标5：在国家期刊上发表摘要。

DESCRIPTION (provided by applicant): This grant is submitted in support of an R-13 application for a non-recurring one day conference entitled: Emerging Cardiac Cellular Therapies: Incorporating cGMP Federal Regulations into Clinical Trials. The Symposium will be held on Thursday September 8, (pm only) and Friday September 9, 2005 (am only) at the Sheraton Inner Harbor Hotel, Baltimore, Maryland. This Symposium is designed to bring together cardiologists, blood stem cell transplanters, researchers and FDA Regulatory Agents from CBER to address the role of the following cell types used in cardiac cellular therapy for treatment of ST Segment Elevation Ml: bone marrow mononuclear cells; mobilized peripheral blood stem cells; enriched subpopulations of cells (i.e., CD34+ or CD133 cells); myoblasts; autologous cardiac stem cells; adipose tissue stem cells; mesenchymal stem cells. These cardiac cell therapy clinical trials must be conducted under the new FDA regulations related to cell therapy processing (cGMPs). To ensure investigator compliance, patient safety and to safeguard the public health, studies involving the use of the cell therapy end product raise these questions regarding current and future clinical trials. What are the latest scientific data behind the use of each cell type?; What clinical results are available for each cell type?; What processing is involved for each cell type?; Are suitable animal models available?; What is the "advertised" function of each cell type in the overall treatment of cardiac damage?; What is the appropriate regulatory framework for each cell type?; What are the biologic/device "combination product" issues that need to be addressed? Aims are: Aim 1: Gather the latest data from cardiac cell therapy clinical trials; Aim 2: Identify laboratory processes involved in various cardiac cell therapy approaches; Aim 3: Identify critical barriers to clinical cell therapy research for cardiac repair and identify pathways around those barriers; Aim 4: Engage cardiac cell therapy investigators in a dialogue about the regulatory framework for their trials; Aim 5: Publish a summary in a national Journal.