Developing of a new approach to clinical trials. —Global clinical trials for ALS and future prospects for Japan—

Abstract

The development of treatments for ALS has been active in recent years, and including pre–clinical trials, there are around 50 clinical trials being conducted worldwide, the most of any neuromuscular disease. In Europe and North America, there is a problem with the large number of clinical trials that ALS patients can participate in, and this has become a new issue in ALS clinical trials called “pipeline pressure”. An attempt to overcome this “pipeline pressure” is being developed by the NEALS (Northwest ALS consortium) in North America. Based on the current situation of ALS clinical trials in Japan, we will introduce the platform trials by NEALS, as well as initiatives for rare cancers and neuroimmunological diseases (CIDP, NMOSD) in Japan, and finally outline the prospects for ALS clinical trials. To avoid the drug loss in ALS in Japan, the key is to consider the establishment of a genetic testing system, the formulation of clinical trial guidelines, and the introduction of adaptive trials that optimize the clinical trial process.