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常住心脏干细胞。这些干细胞不能在心脏病发作（或心肌梗塞）后对心脏进行全面修复。然而，当在实验室中培养并在心肌梗死后给动物时，这些细胞可以启动修复过程，形成新的心肌和新的血管。摩羯座公司有一种培养常驻心脏干细胞的方法，被称为“心球法”。心球的生成可以从一个小的心脏活检开始，可以在门诊过程中获得。该方法正在开发商业化，作为一般心血管疾病的自体治疗。这个项目旨在使心脏球法更快、更便宜、更简单。细胞培养技术将被修改，并通过流式细胞术证明产品的等效性。我们将应用于确定每种细胞产品的功效。该项目还将探索产品银行的可行性，这样患者就可以保存干细胞以备将来使用。在液氮中存放一段时间后，细胞将受到控制速率冻结，然后解冻过程。将采取活力和效力措施，以确定任何有害影响。最后，着眼于未来的临床试验，我们将开发分析方法，使我们能够预测特定患者样本的效力或功效。另一系列动物研究将用于测量心肌梗死的效价，同时将开发一系列简单的实验室效价测定作为候选预测指标。心球干细胞已经进入I/II期临床安全性试验。该项目的具体目标将使Capricor朝着为II/III期临床efo做准备的目标迈进，为疾病提供可能的治疗方法或阻止其进展，并改善国民的健康状况。

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.