Characterization and Potency of Optimized Cardiosphere-derived Stem Cell Method

优化的心球干细胞方法的表征和效力

• 批准号: 8265926
• 负责人: Rachel Ruckdeschel Smith
• 金额: $ 42.54万
• 依托单位: CAPRICOR, INC.
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-01 至 2013-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8265926
• 关键词: Acute; Adipose tissue; Adult; Affect; Allogenic; Animal Model; Animals; Autologous; Biological; Biological Assay; Biological Response Modifier Therapy; Bioluminescence; Biopsy; Biotechnology; Bone Marrow; Cardiac; Cardiac Myocytes; Cardiovascular Diseases; Cause of Death; Cell Culture Techniques; Cell Survival; Cells; Centers for Disease Control and Prevention (U.S.); Clinical; Clinical Trials; Communities; Data Set; Development; Disease; Echocardiography; Endothelial Cells; Engraftment; Ensure; Flow Cytometry; Freezing; Future; Goals; Growth Factor; Healed; Health; Heart; Heart Diseases; Histology; Human; Hypoxia; Immunologics; In Vitro; Infarction; Injection of therapeutic agent; Injury; Ischemia; Left; Legal patent; Leukocytes; Licensing; Liquid substance; Long-Term Effects; Marketing; Measurement; Measures; Medical center; Mesenchymal Stem Cells; Methods; Migration Assay; Modification; Mononuclear; Mus; Myoblasts; Myocardial Infarction; Myocardium; Names; Natural regeneration; Nitrogen; Nutrient; Organ; Outpatients; Patients; Persons; Phase; Pilot Projects; Prevalence; Procedures; Process; Publishing; Recombinants; Research Personnel; Research Proposals; Resistance; Rome; Safety; Sampling; Series; Serum; Simulate; Skeletal Muscle; Source; Starvation; Stem cells; Surface; Technology; Testing; Time; Tube; United States; Universities; Variant; adult stem cell; cell type; commercialization; design; fetal bovine serum; healing; impression; improved; in vivo; manufacturing process; migration; mouse model; paracrine; phase 2 study; potency testing; protein expression; regenerative; repaired; response; safety testing; scale up; success

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.

描述(申请人提供)：最近发现成人心脏含有少量驻留的心脏干细胞。这些干细胞不能在心脏病发作(或心肌梗死)后对心脏进行全面修复。然而，当在实验室培养并在心肌梗死后移植给动物时，这些细胞可以启动修复过程，形成新的心肌和新的血管。Capricor，Inc.有一种培养常驻心脏干细胞的方法，被称为心球法。心球可以从一次小的心脏活检开始，在门诊过程中可以获得。作为心血管疾病的一种自体治疗方法，心球法正在为商业化而开发。该项目旨在使心电圈方法更快、更便宜、更简单。将改进细胞培养技术，并通过流式细胞术证明产品的等效性。ES将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供未来应用。在液氮中储存一段时间后，细胞将受到受控速率的冷冻，然后进行解冻过程。将对生存能力和效力进行衡量，以确定任何有害影响。最后，着眼于未来的临床试验，我们将开发能够预测特定患者样本的效力或有效性的分析方法。将进行另一系列动物研究，以衡量心肌梗死环境中的效力，同时将同时开发一系列简单的实验室效力分析作为候选预测指标。心脏圈来源的干细胞已经被转移到I/II期临床安全试验中。该项目的具体目标将推动Capricor朝着其为第二/第三阶段临床EFO做准备的目标前进，以提供可能的疾病治疗或阻止其进展，并改善国家的健康。