Ethical and Safety Considerations in Stem Cell-Based Therapy for Parkinson’s Disease

Abstract

Stem cell-based therapy for Parkinson’s Disease (PD) is entering an exciting era with many groups competing to reach the goal of safe and practical clinical application. However, the road to this goal is long and beset by challenging obstacles, among which are Good Manufacturing Practice (GMP) standards, scalability, and regulatory requirements for the final cell product. Of paramount importance is the patient safety of the stem cell-derived dopaminergic neurons, such that each stage of the cell therapy implementation process must be scrutinized for potential safety concerns before introduction to the clinic can be contemplated. In this chapter, we will critically consider the safety regulations and safety strategies of stem cell-based therapy for PD, emphasizing the principal requirements necessary for this new therapeutic approach to benefit PD patients. We will introduce the current safety challenges and the connections between these safety issues and the special characteristics of neural stem cells. In addition, we will summarize the safety standards for stem cell-based therapy currently adopted by leading cell therapy groups and international regulations. Both in vitro and in vivo safety assessment methods will be discussed as they relate to the implementation of these standards. Finally, we will speculate on strategies for further enhancing the safety of stem cell-based therapy for PD.