Title of Presentation

“New Era of Medicine with iPS Cells”

Induced pluripotent stem cells (iPSCs) can proliferate almost indefinitely and differentiate into multiple lineages, giving them wide medical application. As a result, they are being used for new cell-based therapies, disease models and drug development around the world.

In 2014, the world’s first clinical study using autologous iPSCs began for the treatment of age-related macular degeneration (AMD). To push these efforts, we are proceeding with a clinical-grade iPSC stock project, which is associated with decreased immune response and less risk of transplant rejection. We started distributing an iPSC stock clone to organizations in Japan, and clinical study using the iPSC stock began for the people with AMD, Parkinson’s disease, Corneal epithelial stem cell deficiency, giving expectation that iPSC-based regenerative medicine will be widely used in the future. Additionally, we reported a gene-editing strategy that could bring iPSC therapies to a wider range of patients. Other applications of iPSCs are drug screening, toxicity studies and disease modeling. In 2017, a new drug screening system using iPS cells for fibrodysplasia ossificans progressiva (FOP) was reported, revealing one drug candidate that goes to clinical trial to treat FOP patients.

In recent years, genome editing technology has made remarkable progress in the medical sciences. This technology has the potential to prevent or treat genetic diseases and gives great hope to patients. On the other hand, there are many technical problems to be solved, such as the possibility of off-target mutation. Furthermore, artificial intelligence (AI) can contribute to medical care and in the US, AI is already being used to predict the risk of heart disease from retinal images and for drug discovery. In order to apply these state-of-the-art technologies to health care, it is mandatory for scientists, government and society to discuss the ethics as well as the efficacy and safety.

Profile

Web Site URL

https://www.cira.kyoto-u.ac.jp/e/index.html

A brief Biography(As of April 1, 2020)

■ Educational History
1987	Kobe University
	School of Medicine (M.D. awarded in March, 1987)
1993	Osaka City University Graduate School
	Division of Medicine (Ph.D. awarded in March, 1993)
■ Professional History
1987	Resident, National Osaka Hospital
1993	Postdoctoral Fellow, Gladstone Institute of Cardiovascular Disease
	The J. David Gladstone Institutes
1996	Assistant Professor, Osaka City University, Medical School
1999	Associate Professor, Nara Institute of Science and Technology
2003	Professor, Nara Institute of Science and Technology
2004	Professor, Institute for Frontier Medical Sciences, Kyoto University
2007	Professor, Institute for Integrated Cell-Material Sciences (iCeMS), Kyoto University
2007 – Present	Professor of Anatomy, University of California
2007 – Present	Senior Investigator, Gladstone Institute of Cardiovascular Disease
	L.K. Whittier Foundation Investigator in Stem Cell Biology
	The J. David Gladstone Institutes
2008	Director, Center for iPS Cell Research and Application (CiRA)
	Institute for Integrated Cell-Material Sciences (iCeMS), Kyoto University
2010	Honorary Professor, Nara Institute of Science and Technology
2010 – Present	Professor and Director, CiRA, Kyoto University

Details of selected Awards and Honors

Nov 2008	Robert Koch Prize 2008
Oct 2009	2009 Albert Lasker Basic Medical Research Award
Oct 2009	2009 Canada Gairdner International Award
Jun 2010	100th Imperial Prize and Japan Academy Prize, The Japan Academy
Nov 2010	26th annual Kyoto Prize in Advanced Technology
Nov 2010	2010 Balzan Prize for Stem Cells: Biology and Potential Applications
May 2011	2011 Wolf Prize in Medicine
Nov 2012	Order of Cultural Merit, Japan
Dec 2012	The Nobel Prize in Physiology or Medicine 2012
Feb 2013	2013 Breakthrough Prize in Life Sciences, Milner Foundation

A list of selected Publications

Mandai, M., Watanabe, A., Kurimoto, Y., Hirami, Y., Morinaga, C., Daimon, T., Fujihara, M., Akimaru, H., Sakai, N., Shibata, Y., Terada, M., Nomiya, Y., Tanishima, S., Nakamura, M., Kamao, H., Sugita, S., Onishi, A., Ito, T., Fujita, K., Kawamata, S., Go, M.J., Shinohara, C., Hata, K., Sawada, M., Yamamoto, M., Ohta, S., Ohara, Y., Yoshida, K., Kuwahara, J., Kitano, Y., Amano, N., Umekage, M., Kitaoka, F., Tanaka, A., Okada, C., Takasu, N., Ogawa, S., Yamanaka, S., Takahashi, M. “Autologous induced stem-cell-derived retinal cells for macular degeneration.” New England Journal of Medicine. 376(11): 1038-1046, 2017

Okita, K., Matsumura, Y., Sato, Y., Okada, A., Morizane, A., Okamoto, S., Hong, H., Nakagawa, M., Tanabe, K., Tezuka, K.-I., Shibata, T., Kunisada, T., Takahashi, M., Takahashi, J., Saji, H., Yamanaka, S. “A more efficient method to generate integration-free human iPS cells” Nature Methods. 8(5): 409-412, 2011

Hong, H., Takahashi, K., Ichisaka, T., Aoi, T., Kanagawa, O., Nakagawa, M., Okita, K., Yamanaka, S. “Suppression of induced pluripotent stem cell generation by the p53-p21 pathway” Nature. 460(7259): 1132-1135, 2009

Nakagawa, M., Koyanagi, M., Tanabe, K., Takahashi, K., Ichisaka, T., Aoi, T., Okita, K., Mochiduki, Y., Takizawa, N., Yamanaka, S. “Generation of induced pluripotent stem cells without Myc from mouse and human fibroblasts.” Nature Biotechnology. 26(1):101-106, 2008

Okita, K., Nakagawa, M., Hyenjong, H., Ichisaka, T., Yamanaka, S. “Generation of mouse induced pluripotent stem cells without viral vectors.” Science. 322(5903): 949-953, 2008

Aoi, T., Yae, K., Nakagawa, M., Ichisaka, T., Okita, K., Takahashi, K., Chiba, T., Yamanaka, S. “Generation of pluripotent stem cells from adult mouse liver and stomach cells.” Science. 321(5889): 699-702, 2008

Takahashi, K., Tanabe, K., Ohnuki, M., Narita, M., Ichisaka, T., Tomoda, K., Yamanaka, S. “Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors.” Cell. 131(5): 861-872, 2007

Okita, K., Ichisaka, T., Yamanaka, S. “Generation of germline-competent induced pluripotent stem cells.” Nature. 448(7151): 313-317, 2007

Takahashi, K., Yamanaka, S. “Induction of Pluripotent Stem Cells from Mouse Embryonic and Adult Fibroblast Cultures by Defined Factors.” Cell. 126(4): 663-676,2006

Mitsui, K., Tokuzawa, Y., Itoh, H., Segawa, K., Murakami, M., Takahashi, K., Maruyama, M., Maeda, M., Yamanaka, S. “The homeoprotein nanog is required for maintenance of pluripotency in mouse epiblast and ES cells.” Cell. 113(5): 631-642, 2003