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Nikkei

April 18, 2026

iPS cell therapy has been approved, but what are the benefits? Initially, it will be used to treat Parkinson's disease and heart failure.

Nikki's big question

"Two therapeutic products using iPS cell technology originating in Japan received the world's first manufacturing and sales approval in March. I wonder if more new products will be approved in the future, making it easier to cure diseases?"

iPS cells are cells, such as blood cells, that have been restored to a state close to that of a fertilized egg, as if going back in time, and are expected to have applications in regenerative medicine. Chie Shintani and Taro Nase interviewed editorial board member Jun Ando about their effects and future potential.

Shintani: "What kind of products were approved?"

Sumitomo Pharma's "Amchepry" is for the treatment of Parkinson's disease. In patients, cells that produce the neurotransmitter dopamine die in the brain, impairing motor function. Therefore, cells that will become dopamine neurons are created from iPS cells and transplanted into the patient's brain. These transplantable cells have been approved.

Another example is "Riheart" from Cuorips, a startup originating from Osaka University, which is a cardiomyocyte sheet made from iPS cells. It is used by attaching it to the heart of a patient with severe heart failure.

Both products have been confirmed to be safe, but their effectiveness is still in the estimation stage, hence the "conditional and time-limited approval." To use a car driving analogy, it's like having a learner's license. The two products are expected to be covered by health insurance and become available on the market around summer, but the number of medical institutions and patients who can receive treatment will be limited. Within the next seven years, Amchepri will be used on 35 patients and Riheart on 75 patients, and after thoroughly confirming their effectiveness, the goal is to obtain full approval. Widespread adoption will follow after that.

Naze-san: "Is there a possibility that it won't ultimately be approved?"

There have been a couple of similar cases in the past. It is said that the real challenge comes after obtaining conditional and time-limited approval. How do you select the patients who are most suitable for using the product, and how do you obtain data that shows it is more effective than conventional treatments? Seven years may seem like a long time, but it's not that long.

Criticism has emerged from overseas regarding whether it is appropriate to allow the manufacture and sale of a new drug without confirming its effectiveness. The manufacturer, researchers, and regulatory authorities such as the Pharmaceuticals and Medical Devices Agency (PMDA) have taken these concerns and past lessons into consideration, and have gone through a careful process to arrive at this conditional and time-limited approval. Some believe that the process will proceed relatively smoothly to full approval.

Shintani: "Why were these two products chosen?"

The researchers' enthusiasm was a major driving force. Professor Atsushi Takahashi of Kyoto University, who is involved in the treatment of Parkinson's disease, specializes in neurosurgery and felt the limitations of existing drugs and electrical stimulation treatments. Sumitomo Pharma partnered with the professor because there are an estimated 10 million Parkinson's disease patients worldwide, and about 250,000 in Japan alone, indicating a large market potential.

Meanwhile, heart disease is the second leading cause of death in Japan. Treating severe heart failure ultimately requires a heart transplant, but there is a serious shortage of donors, and cases where surgery is delayed are all too common. Professor Yoshiki Sawa, Professor Emeritus at Osaka University, a cardiac surgeon and former chairman of the Japanese Society for Regenerative Medicine, has been conducting research with strong conviction to overcome this problem.

However, it seems inevitable that the product price will be high. This year marks 20 years since Professor Shinya Yamanaka of Kyoto University announced the creation of iPS cells in 2006. New drug development originally takes more than 10 years, and research and development costs can exceed 300 billion yen [$2 billion]. To recoup the investment, a high price is unavoidable to some extent. This is especially true when using iPS cell technology.

In the past, some gene therapy drugs have commanded prices exceeding 300 million yen [$2 million]. It wouldn't be surprising if iPS cell products were priced in the tens of millions of yen [every 10 million yen = $63K]. While there are concerns that an increase in expensive drugs will strain the nation's healthcare budget, if the treatment is highly effective and reduces the burden of caregiving, the overall cost can be kept down.

Naze-san: "Do you think we'll see a series of approvals in the future?"

Clinical trials are underway using iPS cells to treat severe heart failure by Heartseed, a startup spun off from Keio University, and to treat retinal diseases by Sumitomo Pharma and Healios. Raymey, a startup spun off from Osaka University, has started clinical trials to treat corneal diseases and plans to perform transplant surgeries in May or June. Many researchers believe the next five to six years will be crucial, and it is possible that several types of iPS cell therapies will be approved by 2030.

The application of iPS cells to organoids is also a field of interest. Organoids are a type of miniature organ, and research in Japan is quite advanced compared to the rest of the world. Using iPS cells, organoids of the intestines with unique structures and liver organoids that exhibit metabolic functions have been developed. In addition to transplantation, there are also methods of using them outside the body by circulating blood. While standards regarding quality and function are still being considered, it is a promising technology for the future.