Clinical trials in the ALS syndrome: it is time for change

What is it about?

Clinical trials in amyotrophic lateral sclerosis have been disappointing since the first positive trial of Riluzole 25 years ago. There are issues as to whether this might reflect errors in trial design rather than drug inefficacy. Trial design in ALS is difficult because the disorder is so variable in its presentation, because it starts long before presentation and because there are difficulties finding sensitive endpoints, rather than death. This editorial reflects on this issues in relation to a report by van Eijk et al analysing the results of 13 earlier trials, published in JNNP.

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