Characterization and Potency of Optimized Cardiosphere-derived Stem Cell Method

描述（由申请人提供）：最近发现成人心脏含有少量的常驻心脏干细胞。这些干细胞不能在心脏病发作（或心肌梗死）后进行全面的心脏修复。然而，当在实验室中培养并在心肌梗死后输送给动物时，这些细胞可以启动修复过程，形成新的心肌和新的血管。Capricor公司有一种培养常驻心脏干细胞的方法，称为心脏球法。可以仅从门诊手术期间获得的小的心脏活检开始生成微球。心脏球方法正在开发中，用于商业化，作为一般心血管疾病的自体治疗。该项目旨在使心脏球方法更快，更便宜，更简单。将对细胞培养技术进行修改，并应用flow cytometry.es证明的产品等效性来确定每种细胞产品的有效性。该项目还将探索产品银行的可行性，以便患者可以保存干细胞用于未来的应用。将细胞进行受控速率冷冻，然后在液氮中储存一段时间后进行解冻。将测量活力和效价，以确定任何有害影响。最后，以未来的临床试验为重点，我们将开发检测方法，使我们能够预测特定患者样本的效力或疗效。将进行另一系列动物研究以测量心肌梗死背景下的效力，同时将平行开发一系列简单的实验室效力测定作为候选预测因子。来自骨髓球的干细胞已经进入I/II期临床安全性试验。该项目的具体目标将推动Capricor实现其准备II/III期临床EFO的目标，为疾病提供可能的治疗方法或阻止其进展，并改善国家的健康状况。

DESCRIPTION (provided by applicant): It was recently discovered that the adult human heart contains small numbers of resident cardiac stem cells. These stem cells are incapable of mounting a full-scale repair of the heart following a heart attack (or myocardial infarction). However, when cultivated in the lab and delivered to animals after a myocardial infarction, these cells can initiate repair processes, form new heart muscle and new vessels. Capricor, Inc. has a method to cultivate resident cardiac stem cells which is known as the cardiosphere method. Cardiospheres can be generated starting with only a small cardiac biopsy that can be obtained during an outpatient procedure. The cardiosphere method is being developed for commercialization as an autologous treatment for cardiovascular disease in general. This project aims to make the cardiosphere method faster, cheaper, and simpler. Cell culture techniques will be modified and product equivalence demonstrated by flow cytometry.es will be applied to determine the efficacy of each cell product. This project will also explore the feasibility of product banking, such that a patient could preserve stem cells for a future application. Cells will be subjected to a controlled-rate freeze followed by a thaw process after a period of banking in liquid nitrogen. Measures of viability and potency will be made to identify any detrimental effects. Finally, with a focus on future clinical trials, we will develop assays that will allow us to predict the potency, or efficacy, of a particular patient's sample. Another series of animal studies will be conducted to measure potency in the setting of myocardial infarction, while a series of simple lab potency assays will be developed in parallel as candidate predictors. Cardiosphere-derived stem cells are already being moved toward a Phase I/II clinical safety trial. The specific aims of this project will move Capricor toward its goal of preparing for a Phase II/III clinical efo provide a possible cure for the disease or halt its progression, and to improve the health of the Nation.