Clinical trials: present and future

The past decade has seen a major expansion of clinical trials in amyotrophi c lateral sclerosis (ALS), However, the perfectly-designed ALS trial remain s elusive. Attempts to track the progression of the disease: are: affected by continual improvements in the care of patients. Comparing the effectiven ess of different drugs is difficult because different primary endpoints are : used in different studies. We also need to decide how much benefit we are aiming to achieve when studying a new treatment. The interpretation of ani mal models has also proved problematic, with results not being replicated i n human studies. Moreover, promising phase I/II trial results have often no t been confirmed by phase III studies, Our patients, meanwhile, are anxious to try any medication that may help, The ALS research community has learne d a great deal from past trials and this will be greatly beneficial when ev aluating the novel and combination therapies currently being developed. Eff ort must also be directed towards the search for objective markers for ALS.