Approval for iPS Cell Treatments: One More Step toward Awaited Practical Application

Editorial

16:32 JST, February 25, 2026

Twenty years have passed since Kyoto University Prof. Shinya Yamanaka succeeded in creating induced pluripotent stem (iPS) cells for the first time anywhere in the world. It is great news that the research, which earned him a Nobel Prize, is finally coming closer to practical application.

The Health, Labor and Welfare Ministry’s expert panel has approved conditional production and sale of regenerative pharmaceutical products derived from iPS cells for treating heart disease and Parkinson’s disease. Given the scarcity of fundamental treatments for both diseases, there must be great hopes among patients and others concerned.

Cardiomyocyte patches developed by a startup originating from the University of Osaka were surgically attached to the hearts of eight patients, resulting in improved exercise function in four. Nerve cells developed by a major pharmaceutical company were transplanted into the brains of six Parkinson’ patients, with symptoms improving in four.

While the patient numbers in both cases are small, and the effects cannot yet be fully confirmed, approval was granted under a scheme for “conditional and time-limited approval” — or early approval — that was established to promote regenerative medicine.

This condition is equivalent to provisional approval, and an application for formal approval must be submitted and granted within the next seven years. Both companies must build up a record of more cases to confirm safety and efficacy.

Early approval is beneficial for promoting technological innovation in drug development and delivering new drugs to patients faster. However, none of the six products that previously received early approval have received formal approval yet.

Regenerative medicine products involve biological material such as cells and genes. Compared to traditional drugs, which can be mass-produced using stable chemicals, they are more prone to variations in quality and are also more difficult to manage. It is also difficult to use them in treatment. Relevant entities should carefully gather data for formal approval.

It was in 2006 when Yamanaka first created iPS cells in mice. Spurred by his winning the Nobel Prize in 2012, the government has consistently allocated substantial budgets over the long term to support this research.

As a result of these efforts, iPS cells are poised to shift from the research stage to practical application. At the same time, the 20-year journey underscores how difficult it is to translate discoveries in basic science into clinical applications.

Emerging companies in Western countries have produced groundbreaking biopharmaceuticals one after another. In Japan, however, an environment in which startups can thrive has not been established, causing the country to fall behind the times.

Yamanaka has said, “The real challenge begins now.” Strengthening collaboration between industry and academia is essential to realize Japan-originated iPS cell medicine. Setting appropriate drug prices for regenerative medicine products, which tend to be expensive, is also important.

(From The Yomiuri Shimbun, Feb. 25, 2026)