JIIP 6th Annual Symposium, 2014: Major InnovaDons – A European PerspecDve Brussels, 17 November 2014 Case study II: Stem cells Daniel Besser Managing Director German Stem Cell Network (GSCN) Berlin, Germany GSCN_pres_slide 1 IntroducDon: Stem cell research – Reality, challenges and perspecDves RegeneraDon of organs High-‐throughput screening Tissue engineering Cell replacement Therapies for an aging society GSCN_pres_slide 2 Photos: stembook.org, Life&Brain, LEBAO, EuroStemCell, Vancouver Sun A network organizaDon supporDng basic and applied stem cell research in Germany Registered NPO associaDon under German law GSCN_pres_slide 3 Goals ! establish an organisaRonal structure for a German network for basic and applied stem cell research ! organize joint annual conferences on stem cell research to be rotated among German ciRes ! launch a plaTorm for communicaRon on scienRfic and strategic issues ! support networking between scienRsts, insRtuRons, private sector, policy-‐makers and the general public Annual Magazine 2013/14: ! foster interacRons between basic and applied stem cell research (translaDon) The GSCN is a member-‐driven iniDaDve of scienDsts, research insDtutes and companies. GSCN_pres_slide 4 What is a stem cell? DefiniDons and hierarchy Hierarchy of stem cells totipotent (zygote) pluripotent (inner cell mass -> embryonic SC or ex-vivo by reprogramming - induced pluripotent SC (iPSCs)) 1. Self-renewal 2. Differentiation hematopoietic SC other somatic stem cells muscle erythrocytes GSCN_pres_slide 5 leukocytes Graphic: stemcells.nih.gov neurons bone other tissues cancer stem cell tumor Reprogramming Yamanaka-‐ Factors GSCN_pres_slide 6 Graphic: LEBAO/MHH Which stem cells for which purpose? SomaDc stem cells in the clinic: • HematopoieDc stem cells (treatment of leukemia) • Skin-‐gra\s for burn vicDms • Cornea regeneraDon with limbal stem cells • Engineering of decellularized Dssues Pluripotent stem cells in clinical trials or upcoming trials: • Pluripotent SC derivaDves for macular regeneraDon in the eye (Japan; London; Adv. Cell Therapies, Inc., MA, USA) • Precursor pancreaDc islet cells in type 1 diabetes (ViaCyte Inc., CA, USA) • SC for Parkinson’s disease • Tissue engineering with iPSCs for heart-‐valve regeneraDon GSCN_pres_slide 7 ! Overall around 4,700 clinical trials using somaDc and pluripotent SC (< 5% hPSCs) (ClinicalTrials.gov) ! 32 facts sheets at EuroStemCell.org on different diseases and related issues The promise of stem cell research Identify drug targets and test potential drugs Study cell differentiation Cultured (pluripotent) stem cells Bone marrow for leukemia & chemotherapy GSCN_pres_slide 8 e.g. Disease modelling, mechanisms of tumorigenesis Tissues/Cells for Transplantation Toxicity testing Nerve cells for Parkinson’s & Alzheimer’s disease Graphic: stemcells.nih.gov Heart muscle cells for heart disease Understanding prevention & treatment of birth defects Pancreatic islet cells for diabetes Routes of commercialisaDon Pharma Academia GMP Clinics GSCN_pres_slide 9 Graphic: stemcells.nih.gov GOOD MANUFACTURING PRACTICE Biotechnology Equipment / material suppliers: cell culture, biologicals, instruments Challenges of stem cell research in Europe • Funding: stem cell research is not idenRfied as own research field but subsumed under content-‐ (e.g. cell biology) or disease-‐driven (cardio-‐ vascular diseases) fields > affects financial resources • Legal: legal framework for stem cell research different in every member state > affects European and internaRonal research co-‐ operaRons • Ethics: differences in ethical aetudes regarding the use of human embryonic stem cells (hESC) and preclinical animal models in EU states > affects social acceptance and funding of research REMEDiE (RegeneraRve Medicine in Europe) project – FP7 hgp://www.york.ac.uk/satsu/remedie/ GSCN_pres_slide 10 Graphic: eurostemcell.org Challenges of stem cell innovaDons in Europe • Funding and knowledge gap to establish standardised protocols and technologies mandatory for preclinical work • High regulatory requirement, lack of experience in regulatory issues, relaRvely new framework “Advanced therapies (ATMP)” • Legal/IP: New key-‐technologies require legal adaptaRons e.g. EUCJ: Brüstle vs. Greenpeace case (2011) – Patentability is excluded for findings based on research with human ESCs • Provider of unproven stem cell therapies GSCN_pres_slide 11 ComparaDve perspecDve EU vs. global approaches • Only member states with more liberal frameworks are involved in iniRaRves (trials) for cell replacement • Lack of a European “stem cell strategy” Global: ! Canadian Stem Cell Strategy & AcRon Plan 2015-‐2025 ! Japanese Research Center Network for RealizaRon of RegeneraRve Medicine (JST – Japan Science & Tech. Agency) ! EU: only naRonal / regional iniRaRves, e.g. Cell Therapy CATAPULT (UK) ! PosiRve excepRon: InnovaRve Medicines IniRaRve (IMI) led to StemBANCC and EBiSC projects – generaRon and banking of hPSCs GSCN_pres_slide 12 ExpectaDons from EU: Funding and support • Recognize stem cell research as separate research field and a unique opportunity to solve upcoming challenges of (an aging) society • Create funding programmes to bridge the gap in the translaRonal process (standardised protocols and processes) • Reduce hurdles / barriers (IP, regulatory requirements) • Encourage a research-‐friendly atmosphere • MoDvate (pharmaceuRcal) companies to cooperate and join research programmes GSCN_pres_slide 13 Acknowledgement • • • • • Ira Herrmann; Stem Cell Network North Rhine Westphalia Stefanie Mahler; German Stem Cell Network Ulrich MarDn; LEBAO, MHH Hannover Albrecht Müller; University of Würzburg EuroStemCell; Edinburgh, Scotland www.eurostemcell.org (supported by H2020 unRl 2017) Further informaDon: • EuroStemCell • REMEDiE (RegeneraRve Medicine in Europe) hgp://www.york.ac.uk/satsu/remedie/ Contact: Dr. Daniel Besser d.besser@mdc-‐berlin.de GSCN_pres_slide 14 CreaDng synergies between all areas of basic and applied stem cell research and providing an interface between science – educaDon – poliDcs – society www.gscn.org GSCN_pres_slide 15
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