Japan Pushes for Industrialization of iPS Cell-Based Medicine with First Drug Application

The practical application of iPS cell-based medicine is approaching. Quolips, a startup originating from Osaka University, has applied to the Ministry of Health, Labour and Welfare for approval of its iPS cell-derived cardiac sheet. Japan, which has been at the forefront of iPS cell research, has lagged behind in commercial development compared to overseas competitors. The global race for iPS cell technology is intensifying, and Japan, as the birthplace of iPS cells, feels the responsibility to lead in practical applications.

iPS cells, capable of differentiating into various cell types and proliferating almost indefinitely, were first created from mice by Professor Shinya Yamanaka of Kyoto University in 2006, followed by human iPS cells in 2007. Japan’s “Act on Ensuring the Safety of Regenerative Medicine” enacted in 2014 accelerated research and development, leading to significant milestones like the world’s first retinal transplant by RIKEN in the same year.

Despite these advances, Japan’s Ministry of Health, Labour and Welfare has been cautious about commercialization, focusing on university-led research. This cautious approach has drawn criticism from pharmaceutical companies and clinicians for lacking a focus on manufacturing technologies and practical application.

In contrast, international companies have been quicker to commercialize iPS cell technologies. For example, Fujifilm-backed Australian company Cynata Therapeutics, US-based Fate Therapeutics, and BlueRock Therapeutics, a Bayer subsidiary, are progressing with clinical trials for various treatments.

At a recent council meeting on regenerative medicine held by the Cabinet Office, it was projected that the market for regenerative medicine products could grow to 10 trillion yen over the next decade. In Japan, startups from Kyoto University and Sumitomo Pharma are conducting clinical trials for Parkinson’s disease, but practical application still seems distant.

If Quolips’ product is approved, it could revitalize Japan’s regenerative medicine industry. The cardiac sheet leverages research by Professor Yoshiki Sawa of Osaka University, who began his work shortly after the breakthrough of iPS cells. Partnering with Terumo, he successfully developed the cardiac sheet for heart treatment.

However, the approval process has just begun, and additional clinical trials may be required. The timing and outcome of the approval remain uncertain. The annual medical cost for heart transplants or artificial heart implants exceeds 10 million yen. The iPS cell-derived cardiac sheet is expected to have a similar or higher price, although provisional approval might result in a lower initial price. While Japan's high-cost medical expense system will limit patient out-of-pocket costs, the overall valuation of the treatment’s novelty and effectiveness will be crucial in determining future investment in the field. This decision will be a pivotal moment for the future of regenerative medicine in Japan.