National Center forResearch 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., MACPDirector,

National Center for Research ResourcesNational Institutes of Health

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 Resourcesthe 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

NCRR

CTSA Consortium Goals: Promoting Efficient Translation from Laboratory to Community

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:

NCRR

Columbia CTSA and Merck Collaboration

Establishment of a seminar series developed to introduce Columbia investigators to drug development by scientists from Merck Pharmaceutical

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:

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

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)

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

NUCATSClinical and Translational Sciences Institute

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 IndustryStanford University

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 IndustryStanford University

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

 

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 IndustryUniversity of California, San Francisco

NCRR

CTSA Translational ProjectsThe 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

NCRR

Providing New Ways to Speed TranslationHarvard 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

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 PortalUC Davis CTSA

NCRR

CTSA Public-Private PartnershipsIntellectual 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/

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 FloridaVIVO: 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

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 BiologyPacific Northwest National Laboratory (PNNL)

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

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.

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

NCRR

Proposal for FY 2012: Creation of theNational 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

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

NCRR

NCATS: Functions

To improve processes in the therapeutics development pipeline by:Experimenting with innovative approaches along the pipeline utilizing an open-access modelChoosing therapeutic projects to test innovative approachesPromoting interactions to advance regulatory science

To catalyze development of novel diagnostics and therapeutics by:Encouraging collaborations, partnerships across all sectorsProviding resources to enable therapeutic developmentEnhancing training in relevant disciplines

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