Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent

Journal: Nature Medicine

Published: 2018-08-20

DOI: 10.1038/s41591-018-0140-5

Affiliations: 9

Authors: 11

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Research Highlight

Parkinson’s disease medication may benefit ALS sufferers

© koto_feja/Getty

© koto_feja/Getty

A medication used to treat Parkinson’s disease could help patients with amyotrophic lateral sclerosis (ALS), according to a new drug screen performed on reprogrammed patient cells.

A Japanese team that included scientists from Tohoku University Graduate School of Medicine generated induced pluripotent stem cells from the blood of patients with ALS — the motor neuron disease that famous physicist Stephen Hawking suffered from.

The researchers grew the stem cells into motor neurons — the brain cells that deteriorate in ALS — and then tested a library of more than 1,200 previously approved drug compounds for agents that would stave off neurodegeneration.

Ropinirole, a drug that activates dopamine receptors in the brain, emerged as the top hit. It helped protect neurons derived from people with both familial and sporadic ALS. The authors are now planning a randomized clinical trial to test ropinirole in ALS patients.

Supported content

  1. Nature Medicine 24, 1579–1589 (2018). doi: 10.1038/s41591-018-0140-5
Institutions FC
Department of Physiology, Keio University, Japan 0.27
Department of Neurology, Nagoya University, Japan 0.23
Department of Neurology, Tohoku University, Japan 0.18
Division of Gene Regulation, Keio University, Japan 0.09
Institute of Medical Sciences, Tokai University, Japan 0.06
Department of Molecular Life Science, Tokai University, Japan 0.06
Research Division of Dementia and Neurodegenerative Disease, Nagoya University, Japan 0.05
Micro/Nano Technology Center (MNTC), Tokai University, Japan 0.03
Research Center for Brain and Nervous Diseases, Tokai University, Japan 0.03

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