Clinical trials: the past, a lesson for the future

摘要

Numerous drugs have been tested in amyotrophic lateral sclerosis with the expectation both that they will treat patients and improve our understanding of some of the basic mechanisms of the disease. Most of these trials were considered to be negative. Careful analysis of the trials does not allow us to clearly discard any of the tested drugs, or any of the suspected mechanisms. Expectations for the future are that: a) we need to be realistic about what to expect; b) we have to define clearly our end-points; c) we have to calculate the expected power before the trials, not after; and d) we have to improve our understanding of the pharmacology.