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Acetylcholine receptors from human muscle as pharmacological targets for ALS therapy

2016; National Academy of Sciences; Volume: 113; Issue: 11 Linguagem: Inglês

10.1073/pnas.1600251113

1091-6490

Eleonora Palma, Jorge Mauricio Reyes‐Ruiz, Diego Lopergolo, Cristina Roseti, Cristina Bertollini, Gabriele Ruffolo, Pierangelo Cifelli, Emanuela Onesti, Cristina Limatola, Ricardo Miledi, Maurizio Inghilleri,

Ion channel regulation and function

Significance Amyotrophic lateral sclerosis (ALS) is a fatal disease leading to motor neuron degeneration and progressive paralysis. Other studies have revealed defects in skeletal muscle even in the absence of motor neuron anomalies, focusing on acetylcholine receptors (AChRs) and supporting the so-called “dying-back” hypothesis. Our results indicate that the endocannabinoid palmitoylethanolamide (PEA) reduces the rundown of AChRs currents in ALS muscle and can clinically improve patients’ pulmonary function. This study strengthens the important role of muscle in ALS pathogenesis and the idea that AChRs can be therapeutic targets.