Using stem cell-derived neurons in drug screening for neurological diseases

Daniel Little; Robin Ketteler; Paul Gissen; Michael J Devine

doi:10.1016/j.neurobiolaging.2019.02.008

Using stem cell-derived neurons in drug screening for neurological diseases

Neurobiol Aging. 2019 Jun:78:130-141. doi: 10.1016/j.neurobiolaging.2019.02.008. Epub 2019 Feb 20.

Authors

Daniel Little¹, Robin Ketteler², Paul Gissen², Michael J Devine³

Affiliations

¹ MRC Laboratory for Molecular Cell Biology, University College London, London, UK. Electronic address: d.little@ucl.ac.uk.
² MRC Laboratory for Molecular Cell Biology, University College London, London, UK.
³ MRC Laboratory for Molecular Cell Biology, University College London, London, UK; Department of Neuroscience, Physiology and Pharmacology, University College London, London, UK.

PMID: 30925301
DOI: 10.1016/j.neurobiolaging.2019.02.008

Abstract

Induced pluripotent stem cells and their derivatives have become an important tool for researching disease mechanisms. It is hoped that they could be used to discover new therapies by providing the most reliable and relevant human in vitro disease models for drug discovery. This review will summarize recent efforts to use stem cell-derived neurons for drug screening. We also explain the current hurdles to using these cells for high-throughput pharmaceutical screening and developments that may help overcome these hurdles. Finally, we critically discuss whether induced pluripotent stem cell-derived neurons will come to fruition as a model that is regularly used to screen for drugs to treat neurological diseases.

Keywords: Alzheimer's disease; Drug discovery; Drug screening; Embryonic stem cells; High content screening; Huntington's disease; Induced pluripotent stem cells; Motor neuron disease; Neurodegeneration; Parkinson's disease.

Publication types

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

MeSH terms

Drug Discovery
Drug Evaluation, Preclinical / methods*
High-Throughput Screening Assays / methods*
Humans
Nervous System Diseases / drug therapy*
Neural Stem Cells*
Pluripotent Stem Cells*

Grants and funding

MC_EX_G0800785/MRC_/Medical Research Council/United Kingdom