Recent Progress in ips Cell Research and Application

Transcription

1 Recent Progress in ips Cell Research and Application Shinya Yamanaka, M.D., Ph.D. Center for ips Cell Research and Application (CiRA), Kyoto University, Japan Takeda - CiRA Joint Program, Shonan, Japan Gladstone Institutes, San Francisco, USA

2

3 Science Overcoming Diseases yrs. 55,000/ Tab ($500/ Tab) By PhD Dre at en.wikipedia, CC BY-SA 3.0, Photo courtesy of Gilead Sciences K.K.

4 The Discovery of ips Cells Oct3/4 Sox2 Klf4 c-myc Skin cells ips Cell (induced Pluripotent Stem cell) Human 2007 Mouse 2006

5 The Features of ips Cells Proliferate infinitely Differentiate into many types of cells (Pluripotency) Neurons Muscle cells Heart muscle cells Blood cells Cartilage cells Pancreatic cells

6 ips Cells

7 eart Muscle Cells

8 The Applications of ips Cells Reprogramming Skin cells Blood cells ips cells Neurons Heart Muscle cells Muscle cells Cartilage cells Regenerative Medicine Drug Development

9 Retinal Cells World s first Transplant in Human (2014) Age-related Macular Degeneration (Photo by RIKEN) Dr. Masayo Takahashi (RIKEN, CDB)

10

11 Autologous ips Cells Transplantation ips cells Differentiated cells Sample collection Quality check Quality check Autograft: Too expensive and time-consuming ips Cell Stock for Regenerative Medicine

12 HLA Homozygous Super Donors HLA homozygous donor

13 Proportion of matched population How Many HLA Homo Donors Are Required? 100% 90% 80% 50% One donor covers ~17% of all the Japanese population! 0% No. of unique HLA-homozygous donors Okita et al., Nature Methods, 2011

14 No. of HLA homo donors How Many Screenings Are Required? th th 50th 6.4 x x x Screening numbers (x 10 4 ) Data provided by Dr. Hiroo Saji

15 ips Cell Stock for Regenerative Medicine Japanese Red Cross Society Platelet / Bone Marrow Donors Informed Consent & Blood Sampling HLA Homozygous Donors Cell Processing Facility (CiRA, FiT) Clinical-grade ips Cells Quality Check Stock Cord Blood Banks Distribution started from 2015

16 Cell Processing Facility At CiRA 2F Passageway 2F Closed Cell Processing Room 1F Inspection Room Containing 8 Cell Processing Rooms

17 ips Cell Stock for Regenerative Medicine ips cells from peripheral blood QHJI (Most frequent HLA):Being distributed RWMH(2 nd most frequent ) :Being distributed DRXT (3 rd most frequent) :SCS production done (SCS; Secondary cell stock) ips cells from cord blood YZWJ (Most frequent HLA) :Being distributed ILCL (Most frequent) :SCS production done GLKV (2 nd most frequent) :SCS production done As of Oct. 13, 2017, 19 donors identified, which would cover ~50% of Japanese population.

18 ips Cell Stock for Regenerative Medicine Distribution as of September 2017 Clinical-grade ipsc 11 projects in 8 institutions and companies Sumitomo Dainippon Pharma Co., Ltd. HEALIOS K.K. Masayo Takahashi Lab (Riken) Yonehiro Kanemura Lab (Osaka National Hospital) Yoshiki Sawa Lab (Osaka Univ.) Keiichi Fukuda Lab(Keio Univ.) Koji Eto, Jun Takahashi, Noriyuki Tsumaki Labs (CiRA), and so on

19 Regenerative Medicine Using ips Cell Stock Neurons Parkinson s Disease ips Cells Retinal / Corneal cells Heart Muscle cells Neural stem cells Pancreatic βcells Immune cells Platelets Cartilage Conducted by Kyoto Univ. & CiRA Blood transfusion Arthritic disorder Technology, ips cells provided by CiRA

20 Clinical Research Using ips Cell Stock HLA matching is useful! March 29, 201 Asahi Shimbun

21 Questionnaire Survey for HLA Matching SEND: 17 user research centers including 3 companies(response rate 100%) July 2016 May 2017 Match or Match if possible increases by 6% from FY2016 to FY2017, which accounts for 50% of the whole in FY2017.

22 Hurdles Donor Recruitment International Collaboration

23 Hurdles Donor Recruitment International Collaboration Cost Patent Regulation

24 Hurdles Donor Recruitment International Collaboration Cost Patent Regulation Regulation Each line: Different Product?

25 ips Cell Stock Members

26 Applications of ips cells Reprogramming Skin cells Blood cells ips cells Neurons Heart Muscle cells Muscle cells Cartilage cells Regenerative Medicine Drug Development

27 Fibrodysplasia Ossificans Progressiva (FOP) Bones formed in muscles, tendons and ligaments 80 patients in Japan (700-1,000 patients worldwide) Healthy person FOP patient

28 Associate Professor Makoto Ikeya

29 Elucidating Disease Mechanism Cartilage FOP patient-derived ips cells Activin A Hino et al., Journal of Clinical Investigation, 2017

30 Drug Discovery using ips cells FOP cells + Activin A FOP cells + Activin A Rapamycin FOP cells + Activin A + Rapamycin Drug Repurposing Hino et al., Journal of Clinical Investigation, 2017

31 The First Clinical Trial for FOP August 2, 2017 Yomiuri Shimbun

32 Collaboration with Takeda Pharmaceutical (T-CiRA Program) CiRA 20 billion yen over 10 years 100 researchers Takeda (each 50 from CiRA and Takeda) Accessible to Takeda s chemical libraries and expertise for drug development 32 Shonan Research Center

33 Thank you for your attention!