Patient and public involvement in amyotrophic lateral sclerosis research in Japan: a survey through nationally advertised ALS Café events and a pilot advocacy training program

Amyotrophic lateral sclerosis (ALS) remains an incurable, progressive neurodegenerative disease. Patient and public involvement (PPI) has been increasingly recognized as a means to enhance the relevance, quality, and efficiency of research; however, its implementation in intractable neurological disease research remains limited in Japan. This webinar-based study, conducted through nationally advertised ALS Café events, aimed to clarify current PPI awareness among the Japanese ALS community and to explore strategies for promoting patient advocacy. Data were collected during the “ALS Café” held in September 2024 and a subsequent patient advocacy training program organized in October 2025. Among 79 respondents (42 patients with ALS and 37 caregivers), approximately 80% reported being unfamiliar with the concept of PPI, yet 72.2% expressed willingness to participate once the concept was explained. Among the 57 respondents willing to participate, interest was highest for involvement in translating research findings into practice (56.1%) and in identifying research themes (47.4%), and most (93%) stated that they expected no remuneration beyond reimbursement of expenses. The subsequent training program was well received, with mean evaluation scores exceeding 8.0 on a 0–10 scale, indicating high comprehensibility among participants (changes in knowledge or willingness to participate were not assessed). Respondents were a self-selected group of ALS Café participants with limited demographic characterization who may not represent patients and caregivers who do not participate in such events, and the training program evaluation lacked a pre-training baseline; these limitations should be considered when interpreting our findings. These findings reveal substantial stated willingness among Japanese patients with ALS and their caregivers to contribute to research despite low baseline awareness of PPI. This gap between limited awareness and high stated willingness suggests that targeted educational efforts could meaningfully expand patient engagement in ALS research. Systematic awareness-building initiatives, structured and accessible engagement opportunities, and sustained collaborative relationships between researchers and the patient community are essential for strengthening patient advocacy and accelerating drug development for ALS in Japan. Amyotrophic lateral sclerosis (ALS) is a serious disease that damages the nerves controlling movement, and there is currently no cure. Researchers are increasingly encouraged to work together with patients and members of the public — an approach known as patient and public involvement, or PPI — to make research more relevant and useful. However, little was known about how familiar Japanese patients with ALS and their families are with PPI, or whether they would want to take part in it. We asked people with ALS and their caregivers who attended an online event called the “ALS Café” in September 2024 to complete a questionnaire about PPI. Of the 79 people who responded, about 80% had never heard of PPI before. Even so, once we explained what PPI means, more than 70% said they would like to be involved in research. Most people wanted to help make sure that research findings are put into practice, and wanted to help choose which questions researchers should study. Most people did not expect to be paid for their time, although many wanted their expenses covered. Based on what we learned, we then created a training program to help patients and caregivers understand ALS research and how they could become involved. We delivered this training in October 2025, and it was very well received, with high understanding scores across all 14 topics covered. Our findings show that although PPI is not yet well known among Japanese patients with ALS, there is strong interest in taking part once it is explained clearly. We believe that ongoing education, together with accessible and low-burden ways to get involved, could help more patients and families take part in ALS research in Japan, which may in turn help speed up the development of new treatments.