Delaying Tactics
A fatal neurodegenerative disease, amyotrophic lateral sclerosis (ALS) causes the progressive loss of motor neurons, cells that send signals from the brain to muscles. Patients gradually lose the ability to move, and ultimately even breathe on their own, often succumbing to respiratory failure. There is no cure, and existing therapies to mitigate symptoms have only limited effects, but new methods of drug discovery offer some hope. Researchers recently reprogrammed induced pluripotent stem cells derived from ALS patients (pictured) to make motor neurons, then used these to identify a potential drug candidate, known as ropinirole hydrochloride. Early clinical trials with small numbers of patients suggest the drug is safe, and that its protective effect on cultured cells translates to real benefits: taking the drug for 48 weeks led to slower declines in motor function, delaying disease progression. More extensive trials will help determine if this promising drug can yield new treatments.
Written by Emmanuelle Briolat
Image from work by Satoru Morimoto and Shinichi Takahashi, and colleagues
Department of Physiology, Keio University School of Medicine, Tokyo, Japan
Image copyright held by the original authors
Research published in Cell Stem Cell, June 2023
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