What is next in ALS clinical trials?

Abstract

Amyotrophic lateral sclerosis (ALS) remains among the most intriguing of the degenerative disorders. Despite is known existence for 150 years, we do not yet understand its pathogenesis. Over the last 20 years, an improved understanding of the genetics of ALS has generated much excitement. The discovery of the Cu,Zn superoxide dismutase gene ( SOD1 ) has ushered in an unprecedented era of ALS research. Transgenic animal models of ALS have been created to study the pathophysiology of the disease. Additionally, these models have been utilized in the screening of potential new therapies, and several agents have appeared effective in these models. This has led to a number of ineffective phase II and III clinical trials. Since the SOD1 mutations represent approximately 2% of all cases of ALS, the relevance of these models to the remaining 98% remains uncertain. This begs the question: are we testing the correct agents or are our trial designs insufficient to prove efficacy? In the present issue, Miller et al. present the results of another unsuccessful clinical trial for the treatment of ALS.1 The present study is the latest of a number of phase II and III clinical trials completed …