Editorial: Can iPS cells clear the barriers to practical use?

It is 20 years since induced pluripotent stem (iPS) cells were first created, a technology that was anticipated to revolutionize regenerative medicine. On August 11, 2006, Kyoto University's Shinya Yamanaka successfully generated iPS cells from mouse skin cells, cells which possess the ability to develop into various tissues and organs.

This groundbreaking work earned Yamanaka the Nobel Prize in Physiology or Medicine in 2012. Recently, on March 2023, two iPS cell-derived products were approved by the Japanese government for the treatment of Parkinson's disease and heart disease. This progress is largely credited to the Japanese government's substantial investment, approximately 110 billion yen over a decade, as well as a policy to expedite the practical application of regenerative medicine.

However, the two approved products currently exist only in a provisional license stage, awaiting further treatment data.

One of the key hurdles facing iPS cells is the complexity of product development. As the treatment relies on living cells, distribution and management present significant challenges, requiring manufacturers to establish robust systems for consistent product quality. Moreover, the side effects of policies aimed at promoting regenerative medicine should not be ignored.

The government enacted the Act on the Safety of Regenerative Medicine in 2014 to establish rules for such treatments. While 6,770 notifications have been submitted, most involve treatments paid for privately, with only a small percentage seeking approval as pharmaceutical products. Many treatments focus on cosmetic concerns and lack substantial scientific backing, even for pain relief or diabetes treatment.

Safety concerns have also arisen, with two patients dying from regenerative medicine treatments for chronic pain and two others suffering serious infections from a cancer prevention treatment.

The Japanese government's substantial investment and policy changes have certainly spurred the practical use of this Japanese technology. Yet, the efficacy and safety of treatments must be thoroughly assessed. The health ministry is currently considering ways to evaluate the appropriateness of treatments, improve the quality of expert review committees, and provide better information to patients.

Despite these efforts, high expectations remain for a technology once considered a "dream treatment." Following Yamanaka's success in creating iPS cells from human skin cells in 2007, numerous patients eagerly awaited practical medical applications. Yamanaka, at the time, expressed a deep sense of responsibility to make this a reality.

The critical question now is whether this research, which has opened a new frontier in biology, can surmount its challenges and ultimately deliver tangible benefits.