Prog Neurobiol. 2018 Apr 10. pii: S0301-0082(17)30104-1. doi: 10.1016/j.pneurobio.2018.04.005. [Epub ahead of print]

Pluripotent Stem Cell-based therapy for Parkinson's disease: current status and future prospects.

Sonntag KC1, Song B2, Lee N2, Jung JH2, Cha Y2, Leblanc P2, Neff C3, Kong SW4, Carter BS5, Schweitzer J6, Kim KS7.

Author information

1

Department of Psychiatry, McLean Hospital, Harvard Medical School, United States; Laboratory for Translational Research on Neurodegeneration, United States; Program for Neuropsychiatric Research, 115 Mill Street, Belmont, MA, 02478, United States.

2

Department of Psychiatry, McLean Hospital, Harvard Medical School, United States; Molecular Neurobiology Laboratory, Program in Neuroscience and Harvard Stem Cell Institute, McLean Hospital, Harvard Medical School, 115 Mill Street, Belmont, MA, 02478, United States.

3

Kaiser Permanente Medical Group, Irvine, CA, 92618, United States.

4

Department of Pediatrics, Harvard Medical School, Boston, MA, 02115, United States; Computational Health Informatics Program, Boston Children's Hospital, Boston, MA, 02115, United States.

5

Department of Neurosurgery, Massachusetts General Hospital, Boston, MA, 02114, United States.

6

Department of Neurosurgery, Massachusetts General Hospital, Boston, MA, 02114, United States. Electronic address: JSCHWEITZER1@MGH.HARVARD.EDU.

7

Department of Psychiatry, McLean Hospital, Harvard Medical School, United States; Molecular Neurobiology Laboratory, Program in Neuroscience and Harvard Stem Cell Institute, McLean Hospital, Harvard Medical School, 115 Mill Street, Belmont, MA, 02478, United States. Electronic address: kskim@mclean.harvard.edu.

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.