Induced Pluripotent Stem Cells for Clinical Use

*Valérie Vanneaux*

## **Abstract**

The use of induced pluripotent stem cells (iPSCs) represents a great promise in regenerative medicine. So far, several clinical trials are underway and preliminary results are promising with the human embryonic stem cells, their non-reprogrammed counterparts. The experience of the clinical use of iPSC derivatives is extremely limited because of several major safety concerns, but many technological advances in the field of iPSC generate high expectations in the near future to develop new clinical trials with an adapted level of patient safety. New guidelines and several recommendations are edited by researchers and regulatory agencies to guarantee the safety of the iPSC products in a clinical context for medical applications. In this chapter, we review the clinical trials with induced pluripotent stem cells and the main factors affecting the safe translation of iPSC to the clinic and how to overcome these issues by standardization and to control the quality of the clinical-grade iPSC products.

**Keywords:** clinical-grade induced pluripotent stem cells, regenerative medicine, clinical trials, quality control

#### **1. Introduction**

Since the discovery of induced pluripotent stem cells (iPSCs) by Yamanaka and Takahashi in 2006, many expectations have emerged, and iPSCs have opened up a world of possibilities for new cell-based therapies in regenerative medicine [1]. In the domain of pluripotent stem cells, iPSCs are considered as equivalent to embryonic stem cells (ESCs), because of two intrinsic key properties: their indefinite proliferative capacities while preserving pluripotency and their capacity to differentiate into all known cell types. However, in contrast to ESCs, iPSCs can be generated without any controversial ethical issues, thus favoring their use in clinical settings. Last but not least, in an autologous approach of cell-based therapy, by using the patient's own cells as source for iPSC generation, one circumvents all the issues related to the immunological compatibility between the donor and receiver. This largely explains the tremendous enthusiasm engendered by iPSC discovery in the sphere of regenerative medicine during the last decade. In this review article, we provide an overview of the launched clinical trials with iPSC and the ongoing efforts to understand the risk related to safety of iPSC-derived cells, highlighting some of the problems that have to be overcome.
