Move Your Research Out of the Ivory Tower and Impact Health: Translating Early-Stage Science to the Market and Improvements in Health

June Lee, MDDirector, Early Translational Research CTSIAssociate Professor, Department of Medicine

Introduction: June Lee

• Joined CTSI on 4/26/11

• Title: Director of Early Translational Research

• Prior experience:– 2004-2011 Genentech Inc

• Head of Infectious Diseases, Cardiovascular/ Metabolic Diseases, Respiratory Diseases in Early Clinical Development

– 1995-2004 UCSF• Pulmonary/Critical Care Faculty

• Director of High Risk Asthma Clinic, SFGH

• Pulmonary Critical Care Fellow

Slide 3

Medical/ Research Training

• 1992-1995 Internship and residency, Internal Medicine

Harbor-UCLA Medical Center

• 1995-1996 Clinical Pulmonary and Critical Care Fellow, University of California, San Francisco

• 1997-2000 Research Postdoctoral Fellow, University of California, San Francisco, Lung Biology Center

• Assistant Professor: University of California, San Francisco (2001-2004), Department of Medicine, Pulmonary and Critical Care Division and Lung Biology Center

• Director: High Risk Asthma Clinic, San Francisco General Hospital, UCSF (2002-2005)

Director of Early Translational Research:Overall Goals

• Enhance/facilitate early research efforts– Facilitate collaborative translational efforts within

UCSF– Facilitate UCSF/industry collaborative efforts– Contribute to early translational research related

educational efforts

Translational Research: Valley of Death

• There is no question that the NIH excels in basic research. What researchers are asking is whether it has neglected the mandate to apply that knowledge. Outside the agency too there is a growing perception that the enormous resources being put into biomedical research, and the huge strides made in understanding disease mechanisms, are not resulting in commensurate gains in new treatments, diagnostics and prevention.

June 2008 | Nature 453, 840-842 (2008) |

How can one encourage fruitful industry-academia collaborations and avoid duplication of R&D?

It's really important to stress that translational work should never be done at the expense of continuing fundamental research. That would be a huge mistake. We cannot slow down our efforts in understanding the behavior of complex biological systems—what I've called the fundamental gap of knowledge. …If you look at the behavior of the industry, it used to be closed-in R&D shops that worked within themselves and really didn't have access to external innovation or external centers of innovation. That is changing for the better. Every company you hear now is saying, “I want to be connected. I want to work with academia. I want to have problems posed to folks who have direct interactions with the diseases themselves in human populations.

Why now?

• UCSF– Leadership– Science/scientists– Will

• External to UCSF– Industry– Funding agencies (NIH)

Translational research in drug development

•Unmet medical

•need/potential clinical

indication(s)

•~18 months

•DISCOVERY •ESR •LSR •ED •ECD (Ph I/II)

•IND submission

and clearance

Early phase drug development activities• Discovery/Early stage research/ Late stage research

– Target selection/validation– Proof of activity– Lead molecule optimization– Evaluation of unmet need

– Define target candidate profile (TCP)

• Late stage research/ Pre-early development– Select lead molecule– Indication (s) selection– Define target product profile (TPP)

• Early Development– Clinical manufacturing– IND enabling toxicology study– Regulatory filing strategy– Overall clinical development strategy

Slide 10

Criteria for Clinical Development• Scientific confidence

– To meet this criterion, a potential medicine must have a solid scientific basis. The proposed mechanism of action must fit current medical understanding and must be supported in laboratory experiments

• Critical medical need– The disease or ailment targeted must be critical and currently

unmet by existing medical technologies. Genentech drops or out-licenses many potential products because they would only marginally improve treatment for a disease or because sufficient means of treatment already exist

• Significant market opportunity

• Adequate market protection

• Reasonable manufacturing economics

Health Benefits, Jobs, &

Products for

Societal Good

Discovery

Bridging the Valley of Death

11

Company Formation

NIHFunding

Seed Funding

Start-up GarageT1

Catalyst

Pfizer Program; VC mentoring

Commercial Funding

Short term work plan

• Ongoing meetings and interviewing of many people around the campus– Translational investigators– Folks involved in addressing challenges of translational

research at UCSF– Other institutions…what works?– Identify the pain points– Continue to build external networks

• Goal– Define key priorities over the next several months

How I and CTSI can help you

• Identify and help facilitate potential collaborations both within UCSF and external to UCSF

• Provide early consultation for projects/programs

• Industry contacts (companies, VCs, experts, etc)

• Potential funds

How you can help me

• Please let me know if what has worked well in the past at UCSF and what could work better

• If you’re aware of successful T1 programs at other CTSAs and other institutions, please let me know

• If you have a project/program/issue that CTSI may be able to help with, please call/contact

• Please contact with any potential external collaborators

• Other inputs/perspectives/constructive feedback are all welcome

What will success look like?

• When an translational research investigator experiences a hurdle, CTSI is a go to source/ partner for help with finding a solution

• Generation of success stories

• Tactics and metrics TBD

Month 1 observations

• UCSF’s core strengths – Brilliant, innovative, talented people– Great discovery science– Deep expertise in many areas– Grassroots problem solving

• Key challenges– Current academic model is not primarily designed

to encourage translational research

Opportunities

• “Better translational research” on the map

• Potential for new and innovative collaborations– With industry partners– Enabled by government funding agencies– Philanthropy

• SF bay area

• Great component parts---UCSF!

T1 Translational Catalyst Program for the Development of Diagnostics and Therapeutics

June Lee, MDDirector, Early Translational Research

Melanie Funes, PhDSOS/T1 Catalyst Program Manager

Juliet FocklerSOS/T1 Catalyst Program Coordinator

T1 Catalyst Program Concept

• Research, technology and programs committed to seeking translational opportunities that may have a potential to develop into a new diagnostic or therapeutic entity, or possibly support identification of a novel diagnostic or therapeutic entity are eligible

• Experts from industry, academia, and venture capital who have experience creating therapies and diagnostic tests will help the researchers outline appropriate next steps for the translation of their discovery and find appropriate partners and funders in industry and foundations. 

T1 Translational Catalyst Program

• Identify the best projects– Diagnostics or therapeutics

• Bring in missing pieces– Panels include people from industry and

academics– Panels review and provide one-on-one feedback

• Leverage limited resources– Identify partnerships

“Phased” Award Approach•Attractiveness•Ease of Implementation•Intellectual Property

Schistosomiasis VIROCHIP

Cartilage Regeneration RunSafe 2 Erythropoietin

Fall 10 AwardLuke – RunSafe Program

Injury Prevention and Performance Improvement

• Multidisciplinary Clinic – Video Gait Analysis– Strength and Flexibility Assessment– Nutritional Analysis and Counseling– Foot Dynamics and Footwear Assessment– Debriefing and Recommendations

• T1 Catalyst support for:– RunSafe in a box– 2D validation– Methodology development– Phased roll out – RunSafe clinic solution

Spring 11 Phase III Candidates

• Brain and Physical Fitness– Integrated physical and cognitive therapeutic software

• CardioPaint– In vivo diagnostic for cardiovascular disease

• Autoimmune disease– In vivo induction of antigen specific Tregs by tolerogenic

vaccination

Solicitations

• Spread the word about T1 Catalyst Program and CTSI

• If you have a program which could potentially benefit from aspects of T1 Catalyst Program, please apply

• Next cycle is in September 2011

http://ctsi.ucsf.edu/funding/t1-catalyst

THANK YOU!

Questions/Comments415-514-8062

june.lee@ucsf.edu