In the past year, Japan has accelerated its position as a hub for regenerative medicine research, largely driven by support from Prime Minister Shinzo Abe who has identified regenerative medicine and cellular therapy as key to the Japan’s strategy to drive economic growth.
The Prime Minister has encouraged a growing range of collaborations between private industry and academic partners through an innovative legal framework approved last fall.
He has also initiated campaigns to drive technological advances in drugs and devices by connecting private companies with public funding sources. The result has been to drive progress in both basic and applied research involving induced pluripotent stem cells (iPS cells) and related stem cell technologies.
“Pretrial Clinical Research” Status in Japan Drives First iPS Cell Clinical Trial in Humans
Indeed, 2013 represented a landmark year in Japan, as it saw the first cellular therapy involving transplant of iPS cells into humans initiated at the RIKEN Center in Kobe, Japan. The RIKEN Center is Japan’s largest, most comprehensive research institution, backed by both Japan’s Health Ministry and government. To speed things along, RIKEN did not seek permission for a clinical trial involving iPS cells, but instead applied for a type of pretrial clinical research allowed under Japanese regulations.
As such, this “pretrial clinical research” allowed the RIKEN research team to test the use of iPS cells for the treatment of wet-type age-related macular degeneration (AMD) on a very small scale, in only a handful of patients. Unfortunately, this trial was “paused” in 2015 due to safety concerns and is currently on hold pending further investigation. Regardless, the trial has set a new international standard for considering iPS cells as a viable cell type to investigate for clinical purposes.
If this iPS cell trial is ultimately reinstated, it will help to accelerate the acceptance of cellular therapies within other countries. Currently, the main concern surrounding iPS cell-based cellular therapy is the fear of creating multiplying cell populations within patients. Even treatments using embryonic stem cells, which have been cultured and studied for decades, are still in very early clinical trials, so it is not surprising that clinical trials using iPS cells are being conducted on a small-scale, experimental level.
Japan has a unique affection for iPS cells, as the cells were originally discovered by the Japanese scientist, Shinya Yamanaka of Kyoto University. Mr. Yamanaka was awarded the Nobel Prize in Physiology or Medicine for 2012, an honor shared jointly with John Gurdon, for the discovery that mature cells can be reprogrammed to become pluripotent.
In addition, Japan’s Education Ministry said it’s planning to spend 110 billion yen ($1.13 billion) on induced pluripotent stem cell research during the next 10 years, and the Japanese parliament has been discussing bills that would “speed the approval process and ensure the safety of such treatments.”
In April, Japanese parliament even passed a law calling for Japan to make regenerative medical treatments like iPSC technology available for its citizens “ahead of the rest of the world.” If those forces were not enough, Masayo Takahashi of the RIKEN Center for Developmental Biology in Kobe, Japan, who is heading the world’s first clinical research using iPSCs in humans, was also chosen by the journal Nature as one of five scientists to watch in 2014.
Japan Becomes Most Liberalized Nation for Development of iPS Cell Products and Services
In summary, Japan is the clear global leader with regard to investment in iPS cell technologies and therapies. While progress with stem cells has not been without setbacks within Japan, including a recent scandal at the RIKEN Institute that involved falsely manipulated research findings and the aforementioned hold on the first clinical trial involving transplant of an iPS cell product into humans, Japan has emerged from these troubles to become the most liberalized and progressive nation pursuing the development of iPS cell products and services.
To learn more, view the “Compete 2015-16 Induced Pluripotent Stem Cell (iPSC) Industry Report.”
 Dvorak, K. (2014). Japan Makes Advance on Stem-Cell Therapy [Online]. Available at: http://online.wsj.com/news/articles/SB10001424127887323689204578571363010820642. Web. 14 Apr. 2015.
 Note: In the United States, some patients have been treated with retina cells derived from embryonic stem cells (ESCs) to treat macular degeneration. There was a successful patient safety test for this stem cell treatment last year that was conducted at the Jules Stein Eye Institute in Los Angeles. The ESC-derived cells used for this study were developed by Advanced Cell Technology, Inc, a company located in Marlborough, Massachusetts.
 Dvorak, K. (2014). Japan Makes Advance on Stem-Cell Therapy [Online]. Available at: http://online.wsj.com/news/articles/SB10001424127887323689204578571363010820642. Web. 8 Apr. 2015.
 Riken.jp. (2014). RIKEN researcher chosen as one of five scientists to watch in 2014 | RIKEN [Online]. Available at: http://www.riken.jp/en/pr/topics/2014/20140107_1/. Web. 14 Apr. 2015.