Luxembourg BioHealth Initiatives

1. Luxembourg BioHealth Initiatives Dr. Jean-Claude Schmit, MD, PhD CEO, Centre de Recherche Public de la Santé (CRP-Santé) President, Integrated BioBank of Luxembourg (IBBL) Prague, RDI Day, 19 October 2009

2. Luxembourg Public Research Landscapein BioHealth Second wave (2003) Third wave (now) First wave (before 1990) IBBL (5→70) Biobanking Cryopreservation EVA (Lippmann) Environment Life sciences (uni.lu) (80) Hospitals CRP-Santé (250) Laboratory research Public health Clinical research PPM Lung cancer LNS/NHL LCSB (2→250) Santec (Tudor) (25) IT & health

3. Biotech SWOT analysis 2005/2006 Health expenditures average 9% of GDP in OECD countries  Health technologies action plan (2007): - leverage existing core competences (research and industrial) - capitalize on Luxembourg’s competitive advantages (central location, international setting, rewarding fiscal environment, dense research network in greater region)  Particular focus on medical devices How did we get there? Why the third wave ?

4. Companies need to cooperate with research institutions in order to fuel the innovation cycle Start-ups or companies that relocate need access to specific incubation spaces Access to financing via dedicated VC funds is crucial: Need to strengthen both volume and quality of research efforts, and link these to economic development Need to concentrate in some core areas of excellence (OECD review of Luxembourg’s innovation system, 2006) Challenges

5. Targeted search for potential strategic partnerships in the United States in the focus area of molecular diagnostics Vision: To establish a research enterprise in the health sciences and technologies and make it grow To attract talents To connect research with healthcare To improve the quality and the efficiency of the health care system To spin off and retain for-profit companies And therewith to also create employment and foster economic diversification Process agreed upon by Government

6. Today: trial-and-error method! Tomorrow: The right drug, for the right person, at the right moment, at a right dosis Personalized medicine is based on molecular diagnostics and requires access to high-quality biological samples (tissue, blood, sputum, etc.) Underlying change of paradigm: the shift from traditional to « personalised medicine »

7. Creation of a Luxembourg Biobank (with TGen, Phoenix Arizona), as an engine to attract and fuel research initiatives Strategic research partnership between the University of Luxembourg and the Institute for Systems Biology (Seattle) Lung cancer research project of CRP-Santé with the « Partnership for Personalized Medicine - PPM» (Phoenix) Three complementary projects

8. IBBL a key infrastructure CRP Santé & Local research capabilities Commercial ventures Hospitals Integrated BioBank of Luxembourg • Sample (patient / non-patient) collection and processing • Sample tracking and distribution • Information collection and analysis Patient CROs Luxembourg research teams European / international research teams ISB (L. Hood) Discovery Project Commercial ventures PPM (L. Hartwell) Demonstration Project Sample collection And analysis R&D New commercialization Health Care applications

9. Integrated Biobank of Luxembourg

10. IBBL’s unique features • Is a nation-wide project, supported by Luxembourg research institutions, hospitals and foreign scientists • Is an independent entity financed by government (60 Mio Euros over 5 years) • Will be built in partnership with international recognised scientists and cover specific research targets • Will support basic-/clinical research and facilitate links to downstream commercial applications • access to samples/data by public and private research (as consented by donor) • clear IP regulations to facilitate cooperation  IBBL is not a “stand-alone” project. It will integrate specific requests of present and future research projects.

11. Leading scientist shaping the Biobank project Dr. Jeffrey M. Trent, PhD President and Scientific Director of T-Gen (Translational Genomics Research Institute) Dr. Trent has worked during 10 years within the National Institutes of Health where he has founded and managed the laboratory charged to coordinate and finalize the Human Genome Project Before his tenure at the NIH, Dr. Trent has been professor at the University of Michigan, where he was the head of the “comprehensive cancer center”

12. IBBL has European / international ambitions IBBL is looking for partners for sample collection (+ is open for customised collections based on specific needs) IBBL is looking for partners for biopreservation research (sample collection/storage, biorefinery, next generation analysis technology) IBBL is a sample provider for public and private research institutions, this includes pharma and biotech companies (selection criteria = scientific value of proposal) Possible collaborations ?

14. II. III. I. Year 1 Year 2 Year 3 Year 4 Year 5 Luxembourg/ISB Strategic Partnership III. Integrated Diagnostic Inc. Seattle Luxembourg II. Blood Organ Fingerprint Project I. Personalized Genome Project

15. Systems Biology: 21st century science Systems biology is the study of an organism, viewed as an integrated and interacting network (bioinformatics) of genes (genomics), proteins (proteomics) and biochemical reactions (metabolomics) which give rise to life. Instead of analyzing individual components or aspects of the organism, such as sugar metabolism or a cell nucleus, systems biologists focus on all the components and the interactions among them, all as part of one system. These interactions are ultimately responsible for an organism´s form and functions.

16. Providing a focus and a vision for biological research in Luxembourg Ambitious research projects to drive innovation Intensive training programs and knowledge transfer Developing a systems biology framework for personalized medicine Creating the framework for new economic activity by setting up the environment for biotechnological research ISB-University projects: Objectives

17. Luxembourg Center for Systems BioMedicine(LCSB) Interdisciplinary Center (IC) of University Overarching topic: systems biology of inflammation Thematic focus: neurodegenerative diseases (others to come) Bioinformatics and advanced mouse models (pre-clinical) to be developed at LCSB National collaborations Genomics (Microarray – CRP-Santé / Next generation sequencing - IBBL) Clinical Proteomics & Metabolomics (Joint unit CRP-Santé and University) Clinical investigation center (CRP-Santé) International collaborations (ESFRI structures) BBMRI (via IBBL), ECRIN (via CRP-Santé), EATRIS & ELIXIR (via LCSB)

18. Dr Lee Hood - Biography Work National Cancer Institute National Institutes of Health 67-70 (Senior Investigator) Caltech-Biology 70-92 (Professor 70-92 and Chairman--80-90) Molecular Biotechnology-UW-92-2000 (Founder and Chairman)-first cross-disciplinary biology department Institute for Systems Biology--2000-present (Co-founder and President)-first systems biology institute Founder and director Founder and director Caltech Microchemical Facility 82-92 Founder and director NSF Science and Technology Center 89-2000 Founder and director Megabase DNA Sequencing Center 96-2003 Co-founder and co-director NanoSystems Biology Alliance (Caltech and UCLA) 2003 to present Co-founder and director Systems Biology Center 2006-present Founder or co-founder-start up companies Founded or co-founded 14 companies, including Amgen, Applied Biosystems Darwin, Rosetta and the Accelerator

19. To be defined Diagnostic companies wishing to develop research results (mainly neurodegenerative diseases) Possible collaborations ?

20. Partnership for Personalized Medicine (PPM)Lung Cancer project Rational behind PPM projects: Define new diagnostics for a specific disease in a timeframe of 3-5 years with the goal of improving treatment and reduce costs Define the disease conjointly with partners (demonstration projects) Test locally the new diagnostic and prove its capacity to diagnose the disease Supervise the patient’s evolution in order to prove the health-enhancing and costs reductions of the treatment

21. Lung Cancer Project: Approximately 180 deaths per year in Luxembourg Approximately 200 newly diagnosed cases per year 40% exhibit metastasis that have a median survival time of 8 months 30% exhibit ganglial metastasis that have a median survival time of 18 months 30% are operable with an median survival time of 3 years Only 8%-15% of all lung cancer patients survive 5 years or more Lung Cancer is at present an almost incurable disease Without a method for improved screening and early detection, it will be difficult to improve lung cancer survival rates Choice of Disease Focus

22. The PPM process

23. US Institutions: Fred Hutchinson Cancer Research Center, Seattle The Biodesign Institute at Arizona State University, Phoenix The Translational Genomics Research Institute , Phoenix Project Lung Cancer: Partnership • Luxembourg Institutions: • CRP-Santé • The National Health Laboratory • Social Security • Central Service for Statistics and Economic Studies (STATEC)

24. Dr. Lee Hartwell - Biography President and director, Fred Hutchinson Cancer Research Center, Seattle (2700 researchers) Professor of genome sciences and adjunct professor of medicine, University of Washington School of Medicine American Cancer Society Research Professor of Genetics Nobel Prize in 2001 in Medicine for his discoveries of the mechanisms of cells division. Page 24

25. Luxembourg aims at setting up, as a complement to the PPM Lung Cancer project, an international network of clinical trial sites for Lung Cancer (TGen has done a similar effort for Pancreatic Cancer) Possible collaborations ?

26. Expected ROIs for Luxembourg Education Research • Provide a well-educated workforce to support both economic and research activities • Create new training / education programs to support research and health care. • Ability to attract top scientists and ramp up existing research programs • Leverage existing competencies • Strong contributions to the education and health care systems. Economy Health care • Research and Education are key assets to attract foreing companies • Existing sectors (IT, logistics) can immediately benefit from the development of research activities • Business failures are less of an issue if the research environment can continuously generate spin-offs and “recycle” both personnel and IP • Health care and research are mutually reinforcing. • Contain health-care costs upstream through personalized therapies and preventive medicine (diagnostics) • A cutting-edge research enterprise facilitates the recruitment of world-class physicians.

27. Personalized medicine is not a new concept, but the technological breakthroughs which make the vision possible are very recent: First mover advantage for Luxembourg Luxembourg’s research institutions are relatively young. They still lack critical mass and international visibility but in the meantime structures are less sclerotic: Opportunity to implement innovative approaches (interdisciplinarity) Why now and why Luxembourg?

28. CRP-Santé: jc.schmit@crp-sante.lu IBBL: robert.hewitt@ibbl.lu LCSB: rudi.balling@uni.lu Contacts