Accelerating development of treatments for amyotrophic lateral sclerosis

Abstract

In May 2023, the Amyotrophic Lateral Sclerosis (ALS) treatment guidelines supervised by the Japanese Society of Neurology were revised for the first time in about 10 years. Riluzole and edaravone are the only two treatment methods (disease–modifying therapy) with established evidence for improving prognosis described therein. Recently, a phase III trial of high–dose methylcobalamin was successful in Japan. Accelerated approval of tofelsen for familial ALS associated with SOD1 gene mutation was granted in the United States, and sodium phenylbutyrate–taurursodiol was approved in the United States and Canada. In Japan as well, there is a demand for the development of a system for conducting clinical trials more rapidly than ever before, and it is important to establish a registry based on patient registration and to understand the natural history of the disease. The Japanese Consortium for Amyotrophic Lateral Sclerosis research (JaCALS) has been established and has already achieved various results. The development of disease models using iPS cells is also progressing, and the development of therapeutic agents associated with it is also progressing. Furthermore, nutritional therapy may also improve the prognosis, and is attracting attention.