The Journal The Authority on Global Business in Japan


December 2013


By Paul E. Cizdziel, Ph.D.

Dr. Shinya Yamanaka, recipient of the 2012 Nobel Prize in Physiology or Medicine, gave a passionate presentation at the Tokyo American Club on October 28. The seminar, “Recent Progress in iPS Cell Research Towards Regenerative Medicine,” attracted 136 people.

Yamanaka spoke about his history, personal philosophy, and promising new applications of induced pluripotent stem cells (iPS). He also explained the enormous potential of iPS cell technology, including implications for cardiac, vision, and neuronal regeneration.

He related how iPS-derived cardiac tissues are being used in drug testing, to determine at an early stage if drug candidates may have toxic side effects. Further, the reconstruction of three-dimensional functioning hearts from stripped tissue scaffolds and iPS-derived cardiomyocytes is showing promise in experimental systems.

According to Yamanaka, the first human iPS cell clinical trials in Japan will soon be underway. The studies are for regeneration of the human retina to correct macular degeneration—a common form of blindness among aging adults. In addition, spinal cord repair using stem cells is showing promise. Researchers have induced mice to regain motor function of their back legs and recover the ability to walk again after spinal cord damage.

Yamanaka discussed how iPS cells are allowing scientists to create unique model systems for studying diseases and the screening of drug candidates, including for the motor neuron disease amyotrophic lateral sclerosis (ALS)—called motor neuron disease (MND) in some countries and Lou Gehrig’s Disease in the United States.

Yamanaka explained that iPS cells made from the cells of ALS sufferers look perfectly normal. However, when induced to become nerve cells, it is clear that the neuronal structure is abnormal—cell dendrites are less than 50 percent the normal length.

Using these ALS-disease model cells, candidate drugs that promote neuronal dendrite growth back to normal length have been identified.

Although the outlook is hopeful for ALS and other diseases where human iPS cell disease model systems are now possible, Yamanaka cautioned that many hurdles remain and additional basic research is needed. The development of potential drugs is many years away.

According to Yamanaka, the level of science and talent in Japan is world-class, and scientists here are clearly capable of making breakthrough discoveries worthy of the Nobel Prize. He was less positive, however, about the translation of research to the clinic for medical application.

Translational research is a key discussion topic in Japan, and is the focus of early-stage plans to establish an organization to facilitate clinical trials and cutting-edge medical care based on the model of the US National Institutes of Health.

Another critical void in Japan, Yamanaka believes, is the lack of CEO-like leadership talent for establishing new and complex fields such as regenerative medicine.

The science, ethics, patents, regulatory, PR, and other considerations and hurdles are numerous, and a special skill set to navigate and organize these requirements is needed. If regenerative medicine is to become a growth driver for the Japanese economy of tomorrow, the commonly employed Japan model of government-sponsored, limited duration special projects to address such issues is probably not sufficient.

The current model is not conducive to develop, attract and retain top visionary talent to build success. Private industry solutions, long-term commitments, and more attractive reward structures are necessary to foster development, create a climate of success, and drive the commercial and medical application of regenerative medicine.

CizdzielDividerPaul E. Cizdziel, Ph.D. is chair of the ACCJ Biosciences Subcommittee. He has 14 years’ management experience in Japan with two leading bioscience companies, and research experience at the RIKEN Yokohama Institute.