Motor neuron disease: urgently needed-biomarkers for amyotrophic lateral sclerosis.

摘要

Since 1996, more than 15 international clinical trials have assessed the efficacy of potential treatments for one of the most aggressive neurological diseases-amyotrophic lateral sclerosis (ALS). The trials were all unsuccessful, and sometimes the trial drugs exacerbated disease progression; therefore, hundreds of millions of euros have, seemingly, been spent in vain. These disappointing trial results are not unique to the ALS field; the vast majority of treatments for Alzheimer disease and stroke that have shown promise in preclinical studies have regularly failed to provide any benefit to patients with these conditions in clinical trials. Most researchers in these disciplines agree that the development of surrogate markers of preclinical and clinical disease might be one means of testing the efficacy of drug interventions more efficiently in the future