TOKYO, JAPAN – People living with ALS and their families in Japan are eager to help shape research on the disease, even though most have never heard of patient and public involvement (PPI), according to a study published on October 30, 2026, in Research Involvement and Engagement.
ALS is a progressive neurodegenerative disease that gradually takes away the ability to move, speak, eat, and breathe, and no cure exists. PPI, the practice of working with patients and members of the public as partners at every stage of research, from choosing research questions to putting results into practice, is increasingly seen as a way to make research more relevant and to speed the development of new treatments. The approach has become well established elsewhere: the United Kingdom has published national standards for public involvement, and North American ALS initiatives train patients as "research ambassadors" and give them an ongoing role in study governance.
Japan has been slower to follow. The Japan Agency for Medical Research and Development (AMED) issued PPI guidance in 2019, but practical implementation and supporting evidence have remained limited, particularly for intractable neurological diseases. The ALS community is now emerging as a driver of change.
A research team led by Professor Osamu Kano of the Toho University Faculty of Medicine, working with collaborators from 16 institutions across Japan, set out to measure where the community stands and to build a path forward. The team used the ALS Café, an interactive forum that Toho University has hosted since 2017 to bring together patients, families, clinicians, researchers, industry, and regulators.
The researchers first surveyed patients and caregivers at an ALS Café webinar held in September 2024 and advertised nationwide through the Japanese Consortium for Amyotrophic Lateral Sclerosis Research (JaCALS), a national ALS patient registry network. The 79 respondents (42 patients and 37 caregivers) answered as follows:
- 79.7% said they did not know what PPI was, and only 8.9% were already involved or knew the concept in depth.
- 72.2% said they would like to take part in PPI once the concept was explained.
- Among those willing to participate, interest was highest in translating research findings into practice (56.1%) and identifying research themes (47.4%).
- Nearly all (98.2%) would agree to a confidentiality agreement, and 93% expected no payment beyond reimbursement of expenses.
Using these results, the team designed a patient advocacy training program and delivered it in October 2025 as a hybrid in-person and online event organized by the Japanese Society of Neurology, with endorsement from the Ministry of Health, Labour and Welfare, AMED, and the Pharmaceuticals and Medical Devices Agency. Some 500 people registered. The program's 14 lectures covered ALS itself, Japan's healthcare and research systems, clinical trials, and expedited access pathways, and every lecture received a mean comprehensibility score above 8 out of 10. Lectures on the healthcare system and on regulatory pathways scored somewhat lower, pointing to where future education should focus.
"To overcome ALS, patients cannot simply wait for research results. I will continue to argue that patients need to take part, side by side with industry, government, and academia," said Professor Kano, the study's corresponding author. "Through patient advocacy, we hope everyone can gain the knowledge to see healthcare as a whole system, and keep discussing together how to make the best use of limited healthcare resources. We hope this momentum will spread to other rare and intractable diseases in Japan."
The authors note that respondents were self-selected participants in ALS Café events and may not represent the wider ALS community, and that the training evaluation had no pre-training baseline. The team plans to establish a standing patient advisory group involved from the design stage of future studies and to track changes in awareness and engagement over time.
This is, to the authors' knowledge, the first report from Japan of a needs-assessment-to-training pipeline for PPI in ALS, delivered through nationally advertised events.
