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iPS cell research and the drive for cures(4) -All 4 episodes-

Prof. Shinya Yamanaka

Photo:Prof. Shinya Yamanaka

Disease modeling and drug screening using iPS cells

Interviewer :
I've heard that iPS cells can be used in drug discovery.

Yamanaka :
Yes, I think in Japan that for both ES and iPS cells there has been too much emphasis on potential uses in regenerative medicine. The target diseases for such approaches are actually rather limited. For diseases that are systemic in nature, spreading throughout the entire body, transplantation does not seem to be a viable option. Drugs, which can be taken orally or injected, are another option with a broad range of uses.
One of the strengths of iPS cells is that they can be derived from patients and used in modeling various pathologies at the cellular level, in what are sometimes called “disease in a dish” studies. It's thought that this type of research will help accelerate the drug discovery process.
In this type of research, iPS cells are simply another tool, and I'm hopeful the technique will be taken up by people who have never used stem cells before and those working in clinical areas. This is already starting to happen in the U.S.

To date, disease modeling has made use of tools such as knockout mice, and I think it's safe to say that just about every lab in the Faculty of Medicine Kyoto University uses these.
But it's important to remember that mice are quite small and only live for around two years. Humans can live 100 years. Genetically, we're different as well, It's known that some drugs that work in mice are ineffective in humans. But with iPS cells we can generate cells that are destined to exhibit some pathology, and therefore study aspects of the disease in vitro. There are limits to this approach, of course. For example, while mice are intact individuals, iPS cells can only generate populations of cultured cells.

The question of how iPS cells could be used to model late-onset diseases, such as ones that take 50 years to be manifested, has been raised, and this is an issue that confronts both iPS cells and knockout mouse research. In the future, we hope to make use of iPS cells generated from patients' tissues, which may help to address this.

Drug discovery is different from clinical research, and is something that could be started immediately. I'm hopeful that even as the technology is in its nascent form, venture businesses will take it up and begin to work with and refine it through trial and error. The problem is that launching a venture company is not easy in Japan, and major pharmaceutical companies are not ready to take action immediately.

Interviewer :
Major pharmaceutical companies are facing the expiration of patents on their proprietary drugs, and there is a move toward generic substitutes, so they seem to begin to change their ways of doing business.

Yamanaka :
Japanese pharmaceutical companies have an R&D budget of several 100 billions of yen, and I'm sure some percentage of that goes to basic research.
Regenerative medicine is certainly one area of interest in iPS cell research, but I'd be happy if people found uses for iPS cells as a tool in drug discovery as well. That may be the quicker path to delivering benefit to patients.
At CiRA, we are also working on deriving patient-specific iPS cells in work led by Professor Tatsutoshi Nakahata. But due to regulations, it isn't easy to supply cell samples from university to industry for further study. The regulatory climate in the U.S. is much more favorable, and I think this helps to widen the development gap. As a new technology, it will take time for iPS cells to gain understanding and acceptance, but I think it will be necessary to build public consensus in the near term.

Interviewer :
Thank you very much for taking the time to speak with us. As you may know, the iPS Trend website has been under the management of the Japan Science and Technology agency (JST), but from July 2010, the site becomes part of the MEXT iPS Cell Research Network, whose secretariat is located in CiRA. We hope that you will continue to have opportunities to publicize the activities and research outcomes of the Network.

Yamanaka :
Yes sure. Thank you. It's been a pleasure.

Photo:A gallery on the 1st floor of the CiRA building open to the public. (Left) The CiRA research building is located at the University Hospital Westcampus.(Right)

A gallery on the 1st floor of the CiRA building open to the public. (Left) The CiRA research building is located at the University Hospital Westcampus.(Right)


Interview by Miwako Homma, supervisor of iPS Trend website, and Katsuaki Sato and Bisei Watanabe from the Japan Science and Technology Agency.

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Shinya Yamanaka, M.D., Ph.D.

Director, Center for iPS cell Research and Application (CiRA), Kyoto University Senior Investigator, Gladstone Institute of Cardiovascular Disease

After graduating from the Kobe University School of Medicine in 1987, and doing his residency at National Osaka Hospital, Yamanaka received his Ph.D. in medical science from Osaka City University Graduate School of Medicine in 1993. He moved to the Gladstone Institute of Cardiovascular Disease in San Francisco that same year as a postdoctoral fellow. He became a Japan Society for the Promotion of Science special postdoctoral fellow in 1996, and an assistant professor in the department of pharmacology at Osaka City University the same year. He moved to the Nara Institute of Science and Technology as associate professor in 1999 and was appointed professor in 2003. He took a professorship at the Institute for Frontier Medical Sciences, Kyoto University in 2004, moving to the Institute for Integrated Cell-Material Sciences (iCeMS) at the same university in 2007, and named director of CiRA,iCeMS Kyoto University in 2008. He is the director of CiRA, Kyoto University since April 2010.

Major Awards

2004 Tokyo Techno Forum 21 Gold Medal (Japan)
2008 Robert-Koch Prize (Germany)
2008 Shaw Prize in Life Science and Medicine (Hong Kong)
2008 Medal of Honor with Purple Ribbon 2008 (Japan)
2009 Canada Gairdner Foundation International Award (Canada)
2009 Albert Lasker Basic Medical Research Award (U.S.)
2010 Imperial Prize of the Japan Academy (Japan)
2012 The Nobel Prize in Physiology or Medicine


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  • MEXT
  • Japan Science and Technology Agency
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