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.
Public Health Relevance Statement: Cardiovascular disease is the No. 1 cause of death in the United States. Cardiosphere-derived stem cells, isolated from a person¿s own heart using Capricor¿s patented technology, can alleviate and reverse many effects of the disease. The cardiosphere technology has the potential to affect many lives, to provide a possible cure for the disease or halt its progression, and to improve the health of the Nation.
NIH Spending Category: Biotechnology; Cardiovascular; Clinical Research; Heart Disease; Heart Disease - Coronary Heart Disease; Regenerative Medicine; Stem Cell Research; Stem Cell Research - Nonembryonic - Human
Project Terms: 21+ years old; Acute; Adipose tissue; Adult; Affect; Allogenic; Animal Model; Animal Models and Related Studies; Animals; Assay; Autologous; Bioassay; Biologic Assays; Biologic Therapy; Biological; Biological Assay; Biological Response Modifier Therapy; Biological Therapy; Bioluminescence; Biopsy; Biotechnology; Blood Serum; Blood leukocyte; Bone Marrow; CDC; Cardiac; Cardiac Diseases; Cardiac Disorders; Cardiac Myocytes; Cardiac infarction; Cardiocyte; Cardiovascular Diseases; Cause of Death; Cell Culture Techniques; Cell Migration Assay; Cell Survival; Cell Viability; Cells; Centers for Disease Control; Centers for Disease Control (U.S.); Centers for Disease Control and Prevention; Centers for Disease Control and Prevention (U.S.); Clinical; Clinical Trials; Clinical Trials, Phase II; Clinical Trials, Unspecified; Communities; Cytofluorometry, Flow; Data Set; Dataset; Development; Disease; Disorder; Echocardiogram; Echocardiography; Effects, Longterm; Endothelial Cells; Engraftment; Ensure; Fatty Tissue; Flow Cytofluorometries; Flow Cytometry; Flow Microfluorimetry; Freezing; Future; GFAC; Goals; Growth Agents; Growth Factor; Growth Factors, Proteins; Growth Substances; Healed; Health; Heart; Heart Diseases; Heart myocyte; Histology; Human; Human, Adult; Human, General; Hypoxia; Hypoxic; Immunologic, Immunochemical; Immunologics; In Vitro; Infarction; Injection of therapeutic agent; Injections; Injury; Investigators; Ischemia; Left; Legal patent; Leukocytes; Licensing; Life; Liquid substance; Long-Term Effects; Mammals, Mice; Man (Taxonomy); Man, Modern; Marketing; Marrow leukocyte; Measurement; Measures; Medical center; Mesenchymal Progenitor Cell; Mesenchymal Stem Cells; Methods; Mice; Microfluorometry, Flow; Migration Assay; Modification; Mononuclear; Mother Cells; Murine; Mus; Muscle Cells, Cardiac; Muscle Cells, Embryonic; Muscle Cells, Heart; Muscle Cells, Precursor; Muscle, Cardiac; Muscle, Heart; Muscle, Skeletal; Muscle, Voluntary; Myoblasts; Myocardial Infarct; Myocardial Infarction; Myocardium; Myocytes, Cardiac; N element; N2 element; Names; Natural regeneration; Nitrogen; Nutrient; Organ; Out-patients; Outpatients; Oxygen Deficiency; Patents; Patients; Persons; Phase; Phase 2 Clinical Trials; Phase II Clinical Trials; Pilot Projects; Prevalence; Procedures; Process; Progenitor Cells; Publishing; Recombinants; Regeneration; Research Personnel; Research Proposals; Researchers; Resistance; Reticuloendothelial System, Bone Marrow; Reticuloendothelial System, Leukocytes; Rome; Safety; Sampling; Series; Serum; Simulate; Skeletal Muscle Tissue; Skeletal muscle structure; Source; Starvation; Stem cells; Surface; Technology; Testing; Time; Transthoracic Echocardiography; Tube; United States; United States Centers for Disease Control; United States Centers for Disease Control and Prevention; Universities; Variant; Variation; White Blood Cells; White Cell; adipose; adult human (21+); adult stem cell; biotherapeutics; biotherapy; cardiac infarct; cardiac muscle; cardiomyocyte; cardiovascular disorder; cell type; clinical investigation; commercialization; coronary attack; coronary infarct; coronary infarction; design; designing; disease/disorder; fetal bovine serum; flow cytophotometry; fluid; healing; heart attack; heart disorder; heart infarct; heart infarction; heart muscle; heart sonography; impression; improved; in vivo; infarct; liquid; manufacturing process; migration; model organism; mouse model; paracrine; phase 2 study; phase 2 trial; phase II trial; pilot study; potency testing; protein expression; protocol, phase II; regenerate; regenerative; repair; repaired; resistant; response; safety testing; scale up; sound measurement; study, phase II; success; white adipose tissue; white blood cell; white blood corpuscle; yellow adipose tissu
