1. National Center for Research Resources NATIONAL INSTITUTES OF HEALTH T r a n s l a t I n g r e s e a r c h f r o m b a s i c d i s c o v e r y t o i m p r o v e d p a t I e n t c a r e Enhancing Translational Research Through Public-Private Partnerships Third Annual Meeting of the Indiana CTSI April 25, 2011 Barbara Alving, M.D., MACP Director, National Center for Research Resources National Institutes of Health

2. NCRR  Improve the way biomedical research is conducted across the country  Reduce the time it takes for laboratory discoveries to become treatments for patients  Engage communities in clinical research efforts  Train a new generation of clinical and translational researchers Led by the National Center for Research Resources the CTSA program supports a national consortium of medical research institutions that work together and share a common vision to: www.ncrr.nih.gov/ctsa Goals of the CTSA Program

3. NCRR CTSA Consortium Goals: Promoting Efficient Translation from Laboratory to Community Promoting Collaborations, Providing Tools, Addressing Informatics NeedsTraining Clinical and Community PracticeClinical ResearchBasic Research Enhancing Health of Communities/ Comparative Effectiveness Research Improving Clinical Research Management Enhancing T1 and Public- Private Partnerships

4. NCRR CTSA Consortium – Building Connections at University of California, Davis  Dynamics of the commercialization process of new inventions  Moving along the path of research to market  Purpose of intellectual property in the commercialization process  When is an idea worth protecting and why  Options for faculty, students, and staff in commercializing their research  Resources available to navigate the journey from research to commercialization Center for Entrepreneurship seminars to explore links between research and inventions. Topics include:

5. NCRR Columbia CTSA and Merck Collaboration  Establishment of a seminar series developed to introduce Columbia investigators to drug development by scientists from Merck Pharmaceutical

6. NCRR Gender Differences in Patenting in the Academic Life Sciences  Lack of exposure to industry contacts  Concern that pursuing commercialization will hinder academic career opportunities  Failure to include patenting into research strategy, especially among senior researchers Ding WW, Murray F, and Stuart TE. Gender differences in patenting in the academic life sciences. Science 2006; 313: 665-667 Reasons for Lower Likelihood for Patenting Among Women Faculty:

7. NCRR Interdisciplinary Team Science at Columbia CTSA  Columbia CTSA pilot funding enabled neurologist Petra Kaufmann, M.D., M.Sc.* to develop an apparatus for children with spinal muscular atrophy (SMA)  Partnered with Elisa Konofagou, Ph.D., Assistant Professor of Biomedical Engineering and Radiology  Outcome: Patent IR 2380: Limb motion capture and rehabilitative assist device * = Currently Associate Director for Clinical Research at the National Institute of Neurological Disorders and Stroke, NIH Gravity Neutral Orthotic (GNO) Device: a novel rehabilitation device for people with severe physical disability

8. NCRR CTSA-Related Translational Efforts at the University of Pennsylvania http://www.itmat.upenn.edu/ctsa/caet/index.shtml  Exploring business practices and best management models in developing collaborations in the pre-competitive space  Developing workgroups to address barriers to collaboration  Engaging both the private and non- profit sector to identify gaps and opportunities for partnership and collaboration with CTSA institutions. Commercialization and Entrepreneurship Program (CAEP)

9. NCRR 2010 CTSA Industry Forum Highlights: Models of Collaboration Between CTSAs and Industry  Students from various disciplines form teams Spend a few weeks in the hospital observing in order to develop lists of clinical needs Develop a need statement based on a market analysis, current products and competition, and customer specifications Brainstorm a technology to solve a problem, screen solutions, develop a prototype, Present it to the companies that sponsor the course Northwestern University: Medical Device Innovation Course NUCATS Clinical and Translational Sciences Institute

10. NCRR Stanford Biodesign Program - mission is to develop leaders in biomedical technical innovation  Yearlong fellowship program for doctors in their residency and graduate engineers  Fellows work in teams and spend two months in clinical immersion to develop a ranked list of 200 unmet needs  Teams then brainstorms solutions and develops implementation strategies that address areas such as IP, regulatory strategy, and clinical trial design Graduate Course in Biodesign  Outside experts (venture capitalists) teach and advise the teams  Teams work on highly-ranked needs that are identified but not pursued by the biodesign fellows  Fifteen companies have already formed based around fellow and student innovation Models of Collaboration Between CTSAs and Industry Stanford University

11. NCRR Stanford  Also has two programs to fund more than $1 million annually in proof-of-concept research:  the CTSA program  Grant from the Wallace H. Coulter Foundation  A request for proposals for the CTSA program received more than 80 proposals, of which 8 were funded Models of Collaboration Between CTSAs and Industry Stanford University

12. NCRR Models of Collaboration Between CTSAs and Industry University of California, San Francisco – T1 Catalyst Program  Brings together academic-industrial teams  Winning proposals work with consultants to make a development plan  Make presentations to potential partners  Activities have resulted in seven funded projects UCSF is testing whether the program could be a model for the entire university

13. NCRR Pfizer Collaboration  $9.5 million, three-year collaboration with Pfizer  Research activities are evenly divided between UCSF and Pfizer  UCSF maintains intellectual property rights to inventions made on its campus, with Pfizer receiving the right to first negotiation  100 proposals have been reviewed and 7 funded  Too early to judge success and the intellectual property negotiations have not yet begun Models of Collaboration Between CTSAs and Industry University of California, San Francisco

14. NCRR CTSA Translational Projects The Scripps Translational Science Institute  CTSA program served as the catalyst that connected Scripps to Qualcomm and other technology firms  Scripps and other CTSAs are conducting a 1,200-patient randomized clinical trial to evaluate a wireless monitor (size of large adhesive bandage) that monitors heart and breathing rates, fluid status, posture, and activity data in patients with chronic heart failure Wireless Health Care Research Scripps Translational Science Institute has partnered with wireless telecommunications company Qualcomm to use wireless technology to potentially decrease health care cost Pictured: Dr. Eric Topol (left), director of the Scripps CTSA and Gary West, a philanthropist

15. NCRR Providing New Ways to Speed Translation Harvard Catalyst & InnoCentive Prize for Innovation February 1, 2010  Harvard launched a search for new solutions for type 1diabetes by posting a challenge on their internal websites  Cash prizes awarded for winning hypotheses or concepts  Harvard investigators will then take these solutions to the next level by conducting experiments to see if they work Using American Recovery and Reinvestment funding Harvard and InnoCentive launched a series of contests aimed at investigating whether new approaches might be useful in sparking novel research directions and collaborations

16. NCRR  Provides investigators with access to “shelved” compounds that can be repurposed for other indications  Has generated strong interest among both researchers and pharma community Pfizer initiated agreement with UC Davis to further develop Portal site UC Davis is also in talks with other industry groups who also wish to list their available compounds http://ctsapharmaportal.org/index.html CTSA Pharmaceutical Assets Portal UC Davis CTSA

17. NCRR CTSA Public-Private Partnerships Intellectual Property Portal  Aggregates and markets technologies from CTSA institutions and NIH  Enhances research activity and private partnerships across the CTSA consortium  Currently there are 15 CTSAs contributing information on their technologies to the site http://www.rochesterctsa.org/ip/

18. NCRR Two Web-based initiatives bring power of Internet-based tools, exemplified by social networking to biomedical research Harvard Medical School eagle-i: Networking Research Resources Across America  Will create a federated national informatics network  9 institutions participating  Goal is to enable any investigator across America to discover research resources that are presently invisible University of Florida VIVO: Enabling National Networking of Scientists  Will create a social network to enable connections among the scientific community and create pathways that lead to others they know  6 institutions participating NCRR Recovery Act Funds to Enhance Scientist and Resource Networking www.ncrr.nih.gov/u24

19. NCRR Collaborative Development of New Technology: NCRR-DOE-Agilent Integration of NCRR and DOE- supported technology into a single platform Collaboration with mass spectrometry instrument company (Agilent) Collaboration with OHSU CTSA : First population scale proteomics experiment Instrumental methods Sample handling Data management and analysis Next Generation Proteomics Platform: Prototype liquid chromatography-ion mobility spectrometry- mass spectrometry (LC-IMS-MS) Proteomics Research Resource Center for Integrative Biology Pacific Northwest National Laboratory (PNNL)

20. NCRR NCRR- fundedTranslational Technology Development: National Resource for Biomedical Accelerator Mass Spectrometry (AMS)  AMS is a highly sensitive technology for metabolic studies  Allows safe microdosing with toxic or experimental molecules in humans  An integrated HPLC-AMS system will make quantitative analysis of metabolism and metabolic networks routine, analysis times will decrease from 36 hours to 30 minutes  Licensed patents to 3 commercial entities  Workshop with Pharma held Nov 19, 2009 Explored uses and barriers to the use of AMS in pharmaceutical research Planning second workshop focused on Academia and CTSAs  Establishing international user’s group  Working with Pharma NIAID contract ($27M/5 yrs) for taking broad-spectrum antibiotics to phase 1 with Trius Therapeutics Access through on fee-for-service ; provides education and training Lawrence Livermore National Laboratory (LLNL) PI: Ken Turteltaub

21. NCRR Thomas Edison: A Design Thinker Design Thinking  Methodology imbues the full spectrum of innovation activities with an understanding of what people want and need Edison’s Approach  Team-based  Multidisciplinary  Good business sense  Nimble budget  Full product launch — light bulb, electric power system, etc. Source: Brown T. Design thinking. Harvard Business Review. June 2008.

22. NCRR Taking a Global View of Design Thinking: Aravind Eye Care System in India Largest and Most Productive Eye Care Center in the World More than an eye hospital, Aravind is:  A social organization committed to the goal of elimination of needless blindness through comprehensive eye care services  An international center for research and implementation of sustainable eye care programs as well as manufacturer of world class ophthalmic products available at affordable costs

23. NCRR Proposal for FY 2012: Creation of the National Center for Advancing Translational Sciences (NCATS) To advance the discipline of translational science and catalyze the development and testing of novel diagnostics and therapeutics across a wide range of human diseases and conditions

24. NCRR NCATS: Challenges & Opportunities  Deluge of new discoveries of potential targets  Unmet therapeutic needs for many conditions, especially rare and neglected diseases  Need to view drug development pipeline as a scientific problem – ripe for experimentation and process engineering

25. NCRR NCATS: Functions To improve processes in the therapeutics development pipeline by:  Experimenting with innovative approaches along the pipeline utilizing an open-access model  Choosing therapeutic projects to test innovative approaches  Promoting interactions to advance regulatory science To catalyze development of novel diagnostics and therapeutics by:  Encouraging collaborations, partnerships across all sectors  Providing resources to enable therapeutic development  Enhancing training in relevant disciplines

26. NCRR NCATS will:  Facilitate – not duplicate – other translational research activities supported by NIH  Complement – not compete with – the private sector  Reinforce – not reduce – NIH’s commitment to basic research