A drug approved for human clinical trials extends lifespan in mice with muscle wasting disease

Scientists have found a way to extend the average lifespan of mice with spinal muscular atrophy (SMA), the leading inherited cause of death in human infants and toddlers. SMA is caused by genetic mutations in survival motor neuron 1 (SMN1). Two years ago, the same research team found that a compound called Y-27632 could increase the lifespan of a certain mouse model by targeting an enzyme that maintains the cellular scaffold. Now, the scientists have shown that another compound, fasudil, works similarly to Y-27632, but is already approved for human clinical trials for other conditions, so it could possibly be tested on humans with SMA sooner. In the study, mice treated with fasudil lived an average of more than 300 days, while untreated mice lived an average of 30.5. However, 300 days is only about half as long as the lifespan of normal mice and while they could perform normal activities better than untreated mice, the treated mice still had a low number of motor neurons.