Induced pluripotent stem cells in Parkinson's disease: scientific and clinical challenges

J Neurol Neurosurg Psychiatry. 2016 Jul;87(7):697-702. doi: 10.1136/jnnp-2015-312036. Epub 2016 Feb 1.

Abstract

Induced pluripotent stem cells (iPSCs), which greatly circumvent the ethical issue of human embryonic stem cells (ESCs), can be induced to differentiate to dopaminergic (DA) neurons, and hence be used as a human disease model for Parkinson's disease (PD). iPSCs can be also utilised to probe the mechanism, and serve as an 'in vivo' platform for drug screening and for cell-replacement therapies. However, any clinical trial approaches should be extensively supported by validated robust biological evidence (based on previous experience with fetal mesencephalic transplantation), in particular, the production and selection of the 'ideal' neurons (functional units with no oncological risk), together with the careful screening of appropriate candidates (such as genetic carriers), with inbuilt safeguards (safety studies) in the evaluation and monitoring (functional neuroimaging of both DA and non-DA system) of trial subjects. While iPSCs hold great promise for PD, there are still numerous scientific and clinical challenges that need to be surmounted before any clinical application can be safely introduced.

Keywords: PARKINSON'S DISEASE.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antiparkinson Agents / therapeutic use
  • Cell Differentiation / physiology
  • Dopaminergic Neurons / cytology*
  • Drug Evaluation, Preclinical
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Induced Pluripotent Stem Cells / transplantation*
  • Parkinson Disease / therapy*

Substances

  • Antiparkinson Agents