1. Johns Hopkins Technology Transfer 1 Translational Biomedical Research: Moving Discovery from Academic Centers to the Community Translational Biomedical Research: Moving Discovery from Academic Centers to the Community Daniel E Ford, MD, MPH David M Levine Professor of Medicine Director, Institute for Clinical and Translational Research Vice Dean for Clinical Investigation Johns Hopkins School of Medicine dford@jhmi.edu

3. Translational Research T1 The process of applying discoveries generated during research in the laboratory, and in preclinical studies, to the development of trials and studies in humans (as well as the process of applying bedside observations to inform laboratory discoveries) T1 The process of applying discoveries generated during research in the laboratory, and in preclinical studies, to the development of trials and studies in humans (as well as the process of applying bedside observations to inform laboratory discoveries) T2 Research aimed at enhancing the adoption of best practices in the community. T2 Research aimed at enhancing the adoption of best practices in the community.

4. Translational Research Activities T1 activities included: 1) the translation of basic discovery into mechanistic studies in cell lines or animals, 2) the translation of mechanistic studies into initial human testing, and 3) the translation of initial human testing into proof of efficacy. T1 activities included: 1) the translation of basic discovery into mechanistic studies in cell lines or animals, 2) the translation of mechanistic studies into initial human testing, and 3) the translation of initial human testing into proof of efficacy.

5. Translational Research Activities The T2 activities included 1) the translation of proof of efficacy into proof of effectiveness in a usual care setting, and 2) research aimed at enhancing the adoption of best practices in the community The T2 activities included 1) the translation of proof of efficacy into proof of effectiveness in a usual care setting, and 2) research aimed at enhancing the adoption of best practices in the community

6. 2010 Survey All faculty and fellows at Johns Hopkins (SOM, SON, SPH, Engineering) All faculty and fellows at Johns Hopkins (SOM, SON, SPH, Engineering) Objectives Objectives proportion of investigators involved in early (T1), late (T2), and reverse translational (RT) researchproportion of investigators involved in early (T1), late (T2), and reverse translational (RT) research barriers to translational researchbarriers to translational research attitudes about translational researchattitudes about translational research involvement in interdisciplinary collaborationinvolvement in interdisciplinary collaboration barriers to collaboration.barriers to collaboration.

7. Results of Survey Majority of researchers indicated they did translational research Majority of researchers indicated they did translational research Highest in engineering facultyHighest in engineering faculty About 60% of basic lab scientistsAbout 60% of basic lab scientists Lower percentage in young researchersLower percentage in young researchers 7

8. Barriers to Translational Research Translational research is not part of my research agenda or is not central to the goals of my research program Translational research is not part of my research agenda or is not central to the goals of my research program I don’t have the resources (funding, space, equipment, technology) for making my research more translational I don’t have the resources (funding, space, equipment, technology) for making my research more translational 8

9. Barriers to Translational Research I have not yet identified an appropriate translational research question to pursue I have not yet identified an appropriate translational research question to pursue I am not sure what the next translational step is I am not sure what the next translational step is I don’t have the training or expertise I need I don’t have the training or expertise I need I have not found the correct collaborators I have not found the correct collaborators 9

10. Mission Statement JHU Institute for Clinical and Translational Research will accelerate improvement in the health of the population by utilizing team driven science to translate basic discoveries into new interventions that optimize health promotion and treatment of disease. JHU Institute for Clinical and Translational Research will accelerate improvement in the health of the population by utilizing team driven science to translate basic discoveries into new interventions that optimize health promotion and treatment of disease. 10

11. Clinical and Translational Sciences Award (CTSA) Program based in the new NIH National Center Advancing Translational Sciences (NCATS) Program based in the new NIH National Center Advancing Translational Sciences (NCATS) Johns Hopkins University is one of 60 Universities with awards Johns Hopkins University is one of 60 Universities with awards http://www.ncats.nih.gov/ http://www.ncats.nih.gov/ 11

12. Translational Research Communities Establish three new Translational Research Communities (TRC’s) - integrated, interdisciplinary, University-wide research and education teams - who will prioritize important clinical problems, develop new technologies and methodologies to solve them, and speed their translation into practice. Establish three new Translational Research Communities (TRC’s) - integrated, interdisciplinary, University-wide research and education teams - who will prioritize important clinical problems, develop new technologies and methodologies to solve them, and speed their translation into practice. 12

13. Translational Research Communities 1. Drugs, Biologics, Vaccines and Devices 2. Biomarkers and Diagnostic Tests 3. Behavioral, Social and Systems Interventions 13

14. ICTR Studio The Studio is both a place and a process, much like a music studio. In simplest terms, The Studio will be a coordinated, multidisciplinary service center with a goal to provide integrated, multidisciplinary research to facilitate translation. The Studio is both a place and a process, much like a music studio. In simplest terms, The Studio will be a coordinated, multidisciplinary service center with a goal to provide integrated, multidisciplinary research to facilitate translation. 14

15. Importance of Data Expand our integrated platform of Cores and Resources with a renewed focus on data. The emergence of “big data” has created new challenges, and success in all research protocols is dependent on the quality and interpretation of data whether “big” or “small”.

16. Johns Hopkins Medicine IRB Approved Protocols 2007 2008 2009 2010 2011 2012 2007 2008 2009 2010 2011 2012 Total New Protocols 1457 1620 1809 2005 2056 2025 Total PIs 944 1011 1081 1215 1248 1247 New RCTs 247 376 405 422 452 459

17. Johns Hopkins Clinical Research Network 17

18. Promising Results So Far 18

19. What do academic researchers need to translate? An ecosystem that includes academic support, federal grant support and commercial partners An ecosystem that includes academic support, federal grant support and commercial partners Believe they can maintain their academic credibility while still commercializing their work Believe they can maintain their academic credibility while still commercializing their work Access to pilot funds Access to pilot funds Access to experts to develop business plans Access to experts to develop business plans 19