ALS治療薬開発に向けた世界の動向と日本の戦略

抄録

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disease, and the development of effective therapies remains a critical global priority. Japan currently has the highest number of approved ALS drugs, including riluzole, edaravone, methylcobalamin, and tofersen. Despite this progress, participation in multinational clinical trials has been limited, raising concerns about potential drug lag and restricted patient access to emerging treatments. Worldwide, approximately 50 ALS trials are ongoing, with innovative designs such as platform and adaptive trials improving efficiency and accelerating therapeutic development.

Several barriers hinder Japan's involvement in global studies, including differences in healthcare systems, regulatory frameworks, and clinical infrastructure. Efforts are underway to establish national clinical trial guidelines aligned with international standards and to strengthen genetic testing networks for familial ALS, thereby improving patient identification and recruitment.

Japan also possesses notable strengths. ALS trials conducted in Japan demonstrate exceptionally low dropout rates, resulting in minimal missing data and high statistical reliability. In addition, the country's compact geography and advanced transportation systems facilitate patient access to trial sites and support adherence. By leveraging these advantages while enhancing patient centralization and promoting earlier engagement in global trial design, Japan can assume a more prominent role in international ALS drug development and help ensure timely access to innovative therapies for patients.