Due dilligence in ALS research
Steve Perrin in a recent issue of NATURE (Vol 507, p.423) summarizes the struggle of the amyotrophic lateral sclerosis (ALS) field to explain the multiple failures of clinical trials testing compounds to improve the symptoms and survival of patients with this disease. He reports the efforts of the ALS Therapy Development Institute (TDI) in Cambridge, Massachusetts, to reproduce the results of around 100 mouse studies which had yielded promising results. As it turned out, most of them, including the ones that led to clinical trials, could not be reproduced, and those where an effect was seen it was dramatically lower than the one reported initially. He discusses a number of measures that need to be taken to improve this situation, all of which have been emphasized independently in other fields of biomedicine where bench to bedside translation has failed.