Case study II - Stem cells Part One JIIP JOANNEUM RESEARCH POLICIES Michael Ploder Angelika Sauer 17th November 2014 17 November 2014 JIIP Symposium 2014 1 STEM CELLS • Stem cells are literally the mother cells of every tissue. • The development of stem cell research over the last centuries opens four major paths of applications 1) Clinical Stem Cell Treatment: Reproduction and Transplantation (Bone marrow/Blood, Skin, Cornea, Cartilage, Fat) 2) Drug Development and Disease Modelling (Differentiated disease models and test systems both for block buster and personalized medicine) 3) (Re) Programming of Tissue Cells (Organ Recreation: Pancreas, Neuronal Syst.) 4) Identification /Treatment of Cancer Stem Cells (Diagnosis, Therapy) 2 Research Landscape critical contributions • The last two decades showed an exponential increase of stem cell research • The research landscape is (increasingly) global USA 1988: Hematopoietic (blood) stem cells from adult mice are purified and characterized USA 1998: The first human 1992:Stem cells are embryonic identified in the adult stem cells human brain are isolated CN 1950 1955 1960 1955: First successful bone marrow transplant USA 1965 1970 1975 1980 1985 1981: Embryonic Stem Cells are isolated from mouse blastocysts UK, USA 1990 1995 2000 2004: The type of nerve cell lost in 2014: Retina sheet Parkinson’s disease made of induced is produced from pluripotent stem cells human embryonic stem cells USA 2005 2010 UK JP USA 2015 2006/7: Induced pluripotent stem cells are discovered 1996:Dolly the sheep, the first mammal to be cloned from an adult stem cell UK JP 2002: Pancreatic cells derived from mouse embryonic stem cells cure diabetes in mice CN 2013: neural organoid generated from hPSC AT 57 196 477 Total costs: Avera ge dura tion (months ): 55 649,4 34 1105,4 44 OF FP-ACTIVITIES: Average (m€): 0,9 SUMMARY OF FP-ACTIVITIES: Pa rtici paproject tionsSUMMARY : size 303 SUMMARY OF FP-ACTIVITIES: 2,2 1277 1,9 1627 EC contribution companies: Countri es i nvolto ved: 9,1% 38 16,1% 41 NUMBERS Stem Stem cellscells Stem cells Projects : 30,7% 24 38 303303 53,7 24 24 425,8 BUDGET EC contribution (m€): EC contribution (m€): BUDGET 39,3% 53,7 55 2,3% 0,9 0,9 27,7% -- Average project (m€): Average size size (m€): Uni vers i ti esproject : 39,3% INSTRUMENTS Companies: PARTICIPANTS PARTICIPANTS RTDvers a ctions 84,5% Uni i ties:: 39,3% EC contribution to companies: Rescontribution ea rch org: 30,7% EC to companies: Compa ni es : 2,3% RTDea knowl edge traUni nsvefer: 14,0% Universities: Res rch org: 30,7% Others : rs i ti e s : 27,7% Research Adoption a nd i nnova 0,0% Re s etion: a rch org: org: RTD a cti ons : 84,5% Compa ni es : 2,3% Companies: RTD knowlni edge tra ns fer: 14,0% Compa e s : Actions to s upportAdopti poloni cy: 1,5% a nd i nnova ti on: 0,0% Others: Othe Others 27,7% Acti onsrsto: s upport pol i cy: 1,5% N/A : 0,0% N/A 0,0% 2,3% COUNTRIES INSTRUMENTS RTD a ctiRTD onsactions: : RTD knowledge transfer:FR RTD knowl e dge tra ns fe84,5% r:FR RTD a ctions : UK Top-5 participating countries Adoption and innovation: Adopti on a nd i nnova ti on:IT (participation activity) UK to support policy: RTD knowl edge traActi nsons fer:Actions 14,0% to s upport pol i cy:DE Top-5 participating countries ES N/A N/A tion: IT Adoption a nd i nnova 0,0% INSTRUMENTS 477 Pa rti ciespa ti ons Countri iParticipations: nvol ved: : Countri eCountries s (m€): i nvol ve d: involved: EC contribution 54Mio 196 Ave ge dura tiduration on (months ): Average (months): Pa rtira ci pa ti ons : 303 Average project size (m€): Others : EC contribution to companies: Total costs: Total EC contribution to costs: companies: BUDGET 57 49,0% 425,8 426Mio Avera ge dura ti on (months ): Compa niproject es : sizeTotal Average (m€): costs: PARTICIPANTS 24 57 57 34 55 55 1277 Proje cts :Projects: NUMBERS Res eacosts: rch org: Total NUMBERS Unicontribution vers i ties : (m€): EC EC Contribution in €: (participation activity) DE Actions to s upport pol i cy: 1,5% Top-5 participating countries Top-5 participating countries ES (participation activity) N/A 0,0% (participation activity) COUNTRIES PARTICIPANTS INSTRUMENTS INSTRUMENTS COUNTRIES • EU-FP has funded Stem Cell Research since the 90‘ Significant increase in FP 6 and FP7 COUNTRIES • PARTICIPANTS BUDGET FP Stem Cell Research COUNTRIES NUMBER BUDGET Projects : Top-5 participating countries (participation activity) FR UK IT DE ES 19644 196 477 341627 34 44 1277 1627 38 41 425,8 890,2 33,0% 649,4 127741 38890,2 12,5% 2,2 1105,4 649,4 53,7 53,7 425,8 2,2 - 9,1% 1,9 5,3% 9,1% 649,4 16,1% 0,90,9 49,0% 2,2 48,5% - 33,0% 12,5% 39,3% 39,3% 5,3% 30,7% 30,7% 77,2% 2,3% 21,2% 2,3% 0,4% 27,7% 27,7% 1,2% 0,0% 84,5% 84,5% 14,0%DE 14,0% UK 0,0% FR 0,0% 1,5% IT 1,5% 0,0% ES 0,0% FR FR UK UK IT IT DE DE ES ES 649,4 1105,4 2,2 77,2% 49,0% 9,1% 28,0% 9,1% 20,5% 21,2% 49,0% 49,0% 33,0% 3,0% 33,0% 33,0% 0,4% 83,0% 12,5% 12,5% 12,5% 12,5% 1,2% 3,0% 5,3% 5,3% 1,4% 5,3% 0,0% 0,0% 12,5% 77,2% UK 21,2% DE 0,4% FR 1,2%IT 0,0%ES 77,2% DE UK 21,2% FR 0,4% DE IT 1,2% UK FR ES 0,0% IT ES DE UK FR IT ES 48,5% 890,2 890Mio 477 44 28,0% 1105,4 1627 41 20,5% 1,9 890,2 3,0% 16,1% 1105,4 1,9 16,1% 48,5% 28,0% 20,5% 3,0% 77,2% 83,0% 12,5% 21,2% 3,0% 0,4% 1,4% 1,2% 0,0% 0,0% UK DE DE UK FR FR IT IT ES ES 1,9 83,0% 48,5% 16,1% 12,5% 48,5% 28,0% 3,0% 28,0% 20,5% 20,5% 1,4% 3,0% 3,0% 0,0% 20,5% 83,0% 12,5% 83,0% UK 3,0% DE 12,5% 1,4% 0,0% FR 3,0% ITUK 1,4% DE ESFR 0,0% IT UKES DE FR IT ES (Currently) 4 Research Landscape actors Hospitals Clinical Laboratories Development of laboratory equipment Equipment, Materials Suppliers Pharma Expansion, differentiation of celltypes, disease modells Clinical Stem Cell Treatment Biobanks, Storage of Patient Samples Medical Faculties/ Universities Life Sciences PROs (Re) Programming of Tissue Cells (Regenerative Medicine) Equipment Suppliers Developing Testing Disease Modelling (Drug development) Biobanks, Storage of Patient Samples Hospitals Clinical Laboratories Tissue collection Equipment, Materials Suppliers Tissue collection Spin offs Biotech Start ups Development of Diagnostics and Therapeutics (VC) Cancer Cancer Treatment Treatment (Personalised (Personalized medicine) medicine) Pharma Hospitals Clinical Laboratories Clinical tests 5 Market development: • Many stem cell treatments are still far from market • Stem cell treatments can save expenses of the clinical health care system and create jobs mainly in academic institutions MARKET READINESS: since Mid 90‘ Clinical Stem Cell Treatment: Reproduction and Transplantation (Skin, Bone marrow, Cornea, Fat Tissue) Drug Development and Disease Modelling (Test Systems) Identification / Treatment of Cancer Stem Cells (Diagnosis, Therapy) (Re) Programming of Tissue Cells (Organ Recreation: Pancreas, Neuronal Syst.) MARKET PLAYERS: CLINICAL LABORATORIES LAB-EQUIPMENT SUPPLIERS 0 to 7 years 7 to 15 years 15 to 40 years PHARMACEUTICAL FIRMS 6 Regulation as competing elements • Development of stem cell research and innovations has been accompanied by an increasing number of different national legislative regulative measures concerning embryonic stem cells – In Germany 2002 the “Stem Cell Act” defined the derivation of human embryonic stem cell (hESC) lines as a criminal act – In UK the Parliament amended the “Human and Embryology Act 1990” to allow research on human embryos for specific purposes. – In Italy clear restrictions of research using embryos are set by the “Law 40” in 2004 – In 2011 the European Court of Justice rules that technologies or inventions based on hESC cannot be patented in the EU (challenge for IPRs and private equity) • 2007 stem cell therapies are classified as “Advanced Therapy Medicinal Products” by the European Commission Regulation 1394/2007 (ATMP) – definition (gene-therapy, somatic-cell therapy, tissue engineering, combined advancedtherapy), authorization • 2008 Establishment of the “Committee for Advanced Therapies” (CAT) located at the European Medical Agency (EMA) – No medicines based on pluripotent stem cells have received marketing authorization in the EU 7 Lessons which might be learned from the stem cell case • Stem cell research and innovation is and will remain a global effort, Biological systems are significantly more complex than engineering based technologies • However local conditions and regulatory and legal frame highly relevant – Institutional Thickness – Regulatory coordination (hESC, preclinical models etc.) – Public awareness • • Stem cell innovation vastly in its infancy Stem cells value chain in public domain to a large extent, (even in the long term) with certain potentials for private economic growth. – high societal and economical impact via clinical treatment, health insurance systems – willingness to pay (private) vs. challenges for reimbursement (public) • FPs could contribute to a significant extent: – – – – Building large consortia (open innovation) including third countries (INCO) Attracting private firms Filling the funding gap between basic research and applied research/application Building ground-work for regulatory development, good practice and co-ordination 8
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