Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease with a rapid clinical course, averaging 4 years from onset to death. In Japan, approximately 12,000 individuals are affected, yet early diagnosis and intervention remain critical challenges. Despite the recent approval of methylcobalamin as an ALS treatment in 2024, Japan lags in global clinical trial participation, with only two out of 11 ongoing phase III trials including Japanese patients. The exclusion from adaptive platform trials raises concerns about “drug loss” wherein new treatments become available overseas but not domestically. To address this issue, the Toho University Omori Medical Center established Japan’s first Northeast ALS Consortium-affiliated ALS clinic in 2017, integrating multidisciplinary care to improve patient outcomes. This “one-stop shop” model provides coordinated services from neurologists, rehabilitation specialists, and genetic counselors, significantly enhancing survival and quality of life. The clinic also pioneered the use of the Hybrid Assistive Limb robotic exoskeleton, demonstrating both short-and long-term benefits in ALS patients’ mobility. Beyond pharmacological and technological interventions, patient and public involvement initiatives, such as the ALS Café, have fostered patient-centered research. These initiatives have shaped studies on tracheostomy decision-making, non-motor symptoms, and disaster preparedness for ventilated patients. To prevent drug loss and advance ALS treatment, Japan must expand its role in global trials, integrate adaptive trial models, and enhance multidisciplinary care. Robotic therapy and patient engagement efforts further reinforce the need for a comprehensive, patient-centered approach. Strengthening these strategies will not only improve treatment accessibility but also contribute to the global fight against ALS.
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