Patient-led research in amyotrophic lateral sclerosis: Quo vadis?

Patient-led trials are steadily claiming their place in modern neurological research. A popular paper on the effects of lithium in amyotrophic lateral sclerosis included a report on a clinical trial in humans. Subsequently, a follow-up study using patient-reported data did not confirm the original result, opening a scientific debate on how patient-reported data could contribute to neurological research. This review critically illustrates the major benefits and caveats of the current patient-led trials and presents recent U.S. and UK guidelines, which aim to the optimal incorporation of patient-reported outcomes in a reinvigorated research perspective.