Induced Pluripotent Stem Cells as a Cell-Based Therapeutic in Stroke

Induced pluripotent stem cells (iPSC) represent a major breakthrough in stem cell research, cell therapy, and regenerative medicine. iPSC have immediate applications in modeling diseases, such as amyotrophic lateral sclerosis, and testing drugs on differentiated human cells in vitro. There is also a growing body of evidence that demonstrates efficacy of iPSC-derived neural progenitor cells in preclinical stroke models in the rodent. Unlike other types of cells such as mesenchymal stem cells that work by immunomodulatory and trophic effects, iPSC work by both “cell replacement” and trophic effects on surrounding tissue. The future clinical use of iPSC-based therapy in regenerative medicine and specifically stroke is still years away and will require overcoming some remaining hurdles. These include the need for safe, nongenomic integrative reprogramming methods to remove the risk of tumor development, choosing the optimal iPSC-derived neural progenitors to transplant, determining the ideal routes, timing, and doses of cell transplantation, and developing good manufacturing practices (GMP) to safely expand and maintain the cells for clinical use. While both autologous and allogeneic transplantations are options, feasibility, cost, and scalability will make allogeneic transplantation the more likely approach.