Kyoto University’s Center for iPS Cell Research and Application (CiRA) and Towa Pharmaceutical Co. have initiated the final phase of clinical trials in May 2025 for a potential Alzheimer’s disease treatment using a repurposed medication, according to a recent announcement.
The research began in 2017 when CiRA scientists, using induced pluripotent stem (iPS) cell experiments, identified that a drug originally developed for Parkinson’s disease could reduce amyloid-beta proteins, which are associated with Alzheimer’s disease onset. Earlier clinical trials demonstrated the drug’s efficacy in patients, paving the way for the current phase three trial, which focuses on individuals with familial Alzheimer’s disease, a rare and rapidly progressing form of the condition.
The trial aims to confirm the drug’s safety and effectiveness in a larger patient group. Professor Haruhisa Inoue of Kyoto University stated, “Repurposing existing drugs could accelerate the delivery of effective treatments to patients.” The team is working toward obtaining regulatory approval as swiftly as possible.
Familial Alzheimer’s disease affects approximately 1% of all Alzheimer’s cases, often striking individuals in their 40s or 50s, according to the Alzheimer’s Association. Japan, with an aging population of over 36 million people aged 65 and older as of 2024 (per the Ministry of Internal Affairs and Communications), faces growing challenges from neurodegenerative diseases. The Ministry of Health, Labour and Welfare estimates that around 4.6 million people in Japan live with dementia, a number projected to rise to 7 million by 2030.
CiRA’s use of iPS cell technology, pioneered by Nobel laureate Shinya Yamanaka, has positioned Kyoto University as a leader in regenerative medicine and drug discovery. Repurposing existing drugs, as in this trial, can significantly reduce development timelines compared to creating new compounds, potentially offering faster relief to patients if successful.
Alzheimer’s disease is a progressive neurodegenerative disorder and the most common cause of dementia worldwide. Japan’s rapidly aging population has made addressing dementia a national priority, with the government launching initiatives like the 2019 National Framework for Promotion of Dementia Policies to improve care and research. The collaboration between CiRA and Towa reflects a broader trend in pharmaceutical research toward drug repurposing, which leverages existing medications to treat new conditions, reducing costs and development time. According to a 2023 report by the Japan Pharmaceutical Manufacturers Association, drug repurposing accounts for approximately 25% of new clinical trials in Japan.
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