Cell Therapy for Alport Syndrome

Abstract

Cell therapy for the treatment of human disease holds much promise, but few to date are standard of care. For regenerative medicine to be practicable, it is essential to have easily accessible sources of stem cells with high therapeutic potential. Advanced stem cell therapeutics aim to correct tissue or organ defects in a targeted manner by supplying stem cells that differentiate into the required cells in situ or are predifferentiated in vitro before infusion. Furthermore, it is critical that the cell preparation be safe and the method of delivery and patient preparation free of complications. In this issue of JASN , LeBleu et al. 1 report on the use of different cell types for the treatment of Alport syndrome that are safe and efficacious. Historically, many stem cell studies have shown efficacy in animals but used systems that were not conducive to clinical application. For example, introduction of genes into cells using viral vectors to trigger differentiation or to correct a mutation are successful in animal models, but their use in humans remains a source of concern.2 Similarly, for human embryonic stem (ES) cells to be used in patients, hurdles such as tissue matching, sourcing of cells, and the risk for teratomas need to be overcome. Some of these hurdles are circumvented by the production of induced pluripotent stem cells using removable vectors3 and the development of methods to prevent teratoma formation.4 Although many cell therapy studies are not suitable for clinical use, they are important to our understanding of stem cells and their role in treating disease. To date, the only stem cells used therapeutically are hematopoietic stem cells for bone marrow transplantation. Cell-based therapies for the treatment of kidney disease have not been explored extensively. The current treatment for …