The Journal The Authority on Global Business in Japan


JULY 2014
Open Door for Stem Cell Research
New clinical trial guidelines could stimulate major medical advances

By Paul Cizdziel, PhD; Michael Alfant; and Bryan Norton

Nobel Prize laureate Dr. Shinya Yamanaka recently visited the Embassy of the United States in Tokyo to discuss innovation and medical science.

Yamanaka is a professor at Kyoto University and director of its Center for iPS Cell Research and Application (CiRA). He spoke at an ACCJ luncheon last October.

Accompanying Yamanaka was Dr. Kentaro Azuma, associate professor and chief administrator for the Medical Applications Promotion Office at CiRA.

The meeting on May 16 was arranged by ACCJ leaders to highlight particular topics certain to have a global impact on innovation and the commercialization of regenerative medicine and other medical technologies.

Yamanaka has been an outspoken proponent for policy changes that would lengthen allowable project timelines for research grants. This would encourage greater exploration of daring theories and ideas under the banner of maximizing innovation and discovery.

Yamanaka lamented how short-term funding commitments usually require researchers to demonstrate incremental progress. This may be safe and practical, but it is insufficient to encourage visionary quantum leaps in understanding or major scientific advances.

Yamanaka’s own research to discover induced pluripotent stem (iPS) cell technology is one example of a risky long-term project, which eventually led to him receiving the 2012 Nobel Prize in Physiology or Medicine.

This year will engender a bold initiative by the Ministry of Health, Labour and Welfare to facilitate clinical application of stem cell-related or other regenerative medicine therapies.

The new guidelines for clinical trials and conditional marketing approvals have recently been opened for comment, and are expected to be passed into law later this year.

Due to the cost, ethical considerations, and customized nature of the medical procedures, the classic placebo model blind-study clinical trials—used for small-molecule drugs—are considered untenable for commercial progress of regenerative medicine.

Azuma, who previously worked at the ministry, explained the new model and the proposed three-stage risk classification system.

Discussion ensued about the need for harmonization of clinical regulations—and exemptions—between leading nations such as the United States and Japan, which are at the forefront of stem cell-related research. Regenerative medicine is still in a very early stage with regard to medical practice, so the timing is right for synchronization.

Unfortunately, cumbersome hurdles to that harmonization exist, including the fact that the 178-page proposed legal guidelines, now open for comment (, have only been released in Japanese.

This makes it difficult to garner international feedback through advocacy organizations such as the ACCJ.

The need for international perspectives that draw on global best practices is especially important, since the proposed legislation is expected to create a clinical trial framework that is progressive and attractive to American (and other foreign) companies to launch regenerative medicine technology in Japan, as a possible first choice market.

These topics and others, such as the under-utilization of female scientific researchers in Japan and current incentive programs for investment in the United States (e.g., the SelectUSA initiative), were also discussed.

Yamanaka had the chance to greet Ambassador Caroline Kennedy in her office after the meeting.

These meetings are expected to be a prelude to further dialogue among CiRA, embassy officials, and ACCJ leaders, in the hope of facilitating cooperative initiatives for scientific innovation and the advancement of regenerative medicine between the United States and Japan. •