Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent
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.
- Nature Medicine 24, 1579–1589 (2018). doi: 10.1038/s41591-018-0140-5
|Keio University, Japan||0.36|
|Nagoya University, Japan||0.27|
|Tokai University, Japan||0.18|
|Tohoku University, Japan||0.18|