Cell Therapy and Heart Failure

Orthotopic heart transplantation is the only curative therapy for advanced heart failure. With a limited supply of donor organs, other therapies are being explored, including cellular replacement. Somatic cell therapy (SCT) has been used effectively for other chronic diseases, such as bone marrow transplantation to treat certain cancers and many other diseases including allotransplantation of pancreatic islets for patients with life-threatening complications from diabetes mellitus. This chapter focuses on using somatic cell therapy for patients with cardiovascular disease (including heart failure)—describing the types of available cells and their benefits and limitations, procedures, and the results and implications of clinical studies. Research has showed mixed outcomes for SCT and modest beneficial effects, but these results may be attributed to differences in the types of cells used (CD34+, bone marrow mononuclear, mesenchymal stem, etc.), cell processing and storage, mode of delivery, patient selection, and cell source (autologous or allogeneic). With limited cardiovascular functional response but a continuing need for effective treatment and to learn more definitively the extent of promise for SCT’s role in treating heart failure, research with well-defined primary and secondary end points is required to determine whether SCT reduces morbidity and improves survival in patients with cardiomyopathy. SCT for the treatment of heart failure may have an impact other than remuscularization including reverse remodeling of the failing heart, modulation of the fibroproliferative response following injury, vasculogenesis, or mobilization of endogenous cardiac progenitors. One ongoing source of collaborative research is the Cardiovascular Cell Therapy Research Network, funded by the National Heart, Blood, and Lung Institute. These and other investigators are researching factors such as the optimal cell population and cell preparation, mechanisms of action of cell therapy, the role of multiple or serial interventions with cell delivery, and identification of the patients who could benefit most from SCT. These clinical SCT studies will continue to benefit from the mechanistic studies undertaken in the basic science laborato ries.