Pluripotent stem cell-based therapy for Parkinson's disease: Current status and future prospects


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Abstract

Parkinson's disease (PD) is one of the most common neurodegenerative disorders, which affects about 0.3% of the general population. As the population in the developed world ages, this creates an escalating burden on society both in economic terms and in quality of life for these patients and for the families that support them. Although currently available pharmacological or surgical treatments may significantly improve the quality of life of many patients with PD, these are symptomatic treatments that do not slow or stop the progressive course of the disease. Because motor impairments in PD largely result from loss of midbrain dopamine neurons in the substantia nigra pars compacta, PD has long been considered to be one of the most promising target diseases for cell-based therapy. Indeed, numerous clinical and preclinical studies using fetal cell transplantation have provided proof of concept that cell replacement therapy may be a viable therapeutic approach for PD. However, the use of human fetal cells as a standardized therapeutic regimen has been fraught with fundamental ethical, practical, and clinical issues, prompting scientists to explore alternative cell sources. Based on groundbreaking establishments of human embryonic stem cells and induced pluripotent stem cells, these human pluripotent stem cells have been the subject of extensive research, leading to tremendous advancement in our understanding of these novel classes of stem cells and promising great potential for regenerative medicine. In this review, we discuss the prospects and challenges of human pluripotent stem cell-based cell therapy for PD.HighlightsProspects and challenges of human pluripotent stem cell-based cell therapy for PD.Revisiting the concept of personalized cell replacement therapy for PD using iPSCs-derived DA cell populations as an autologous cell source.Lessons learned from clinical trials using fVM cells, and ongoing pre-clinical studies using hESCs, and hiPSCs.Critical steps required to generate clinical grade iPSCs and derivation of functional DA cell populations for transplantation.Clinical roadmap for personalized cell therapy identifying obstacles that must be overcome.

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