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: 7745381
• 负责人: Rachel Ruckdeschel Smith
• 金额: $ 12.48万
• 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 2011-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7745381
• 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; Life; 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; Phase II Clinical Trials; 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; 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（由申请人提供）：最近发现，成年人类心脏含有少量的居民心脏干细胞。这些干细胞无法在心脏病发作（或心肌梗塞）后对心脏进行全面修复。但是，当在实验室中培养并在心肌梗塞后将其送到动物时，这些细胞可以启动修复过程，形成新的心肌和新容器。 Crapricor，Inc。有一种培养居民心脏干细胞的方法，称为心圈方法。可以从门诊手术期间获得的小心脏活检开始，可以产生心层。总体上，正在开发用于商业化作为一种​​自体疗法的商业化方法。该项目的目的是使心界圈方法更快，更便宜，更简单。细胞培养技术将进行修改，并通过流式细胞仪证明的产物等效性。E将应用于确定每个细胞产物的功效。该项目还将探索产品银行业务的可行性，以便患者可以为将来的应用保留干细胞。细胞将经过受控的率冻结，然后在液体氮的储存期后进行融化过程。将采取可行性和效力的措施来确定任何有害影响。最后，以未来的临床试验为重点，我们将开发测定法，使我们能够预测特定患者样本的效力或功效。将在心肌梗死的情况下进行另一系列动物研究，以测量效力，而一系列简单的LAB效力分析将以候选预测指标并行开发。心圈衍生的干细胞已经转移到I/II期临床安全试验中。该项目的具体目的将使交换机朝着为II/III期临床EFO做准备的目标，可以治愈该疾病或停止其进展，并改善国家的健康状况。