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

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.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
Project #
4R44HL095203-02
Application #
8038065
Study Section
Special Emphasis Panel (ZRG1-CVS-K (10))
Program Officer
Buxton, Denis B
Project Start
2009-09-01
Project End
2013-02-28
Budget Start
2011-03-01
Budget End
2012-02-29
Support Year
2
Fiscal Year
2011
Total Cost
$397,217
Indirect Cost
Name
Capricor, Inc.
Department
Type
DUNS #
625221820
City
West Hollywood
State
CA
Country
United States
Zip Code
90048