Exceptional Opportunities in Biomedical Research

Francis S. Collins, M.D., Ph.D. Director, National Institutes of Health Virginia Summit on Science, Engineering, & Medicine November 15, 2013

NIH: Steward of Medical and Behavioral Research for the Nation
“Science in pursuit of fundamental knowledge about the nature and behavior of living systems ... and the application of that knowledge to extend healthy life and reduce illness and disability.”

NIH Extramural & Intramural Funding
FY 2013 Full-Year CR Level: $29.1 Billion
Spending at NIH

Spending Outside NIH

Physiology or Medicine 2013

NIH-Supported Nobel Prize Awardees
Photo: © Yale University Photo: H. Goren © HHMI Photo: © S. Fisch

James E. Rothman

Randy W. Schekman Thomas C. Südhof

Chemistry 2013
144 NIH-supported researchers have become Nobel Laureates

© Harvard University

Photo: © S. Fisch

Photo: Wikimedia Commons

Martin Karplus

Michael Levitt

Arieh Warshel

NIH’s Impact on U.S. Health and Medicine U.S. Life Expectancy
80 78 76 74 72 70 68
1970 1978 1986
1972 1974 1976 1980 1982 1984 1988 1990 1992 1994 1996 1998 2000 2002 2004 2006 2008 2010

Life expectancy gains worth ~$3.2 trillion annually

66

NIH Accomplishments
Reduction in deaths from:
 Heart disease  Stroke  HIV/AIDS
Cardiovascular disease death rates have fallen > 60% in the last half-century

Increased survival rates for:
 Breast cancer  Cervical cancer  Colon cancer
Cancer rates now falling ~1% per year; each 1% drop saves ~$500 B

HIV therapies enable people in their 20s to live to age 70+

NIH Funds Scientists Across U.S.

Alaska

Hawaii

NIH Funding in Virginia (FY2013) $272,431,861

Impact of NIH-supported Research on U.S. Economy
 In 2012, NIH funding
– Supported >402,000 jobs at 2500 institutions, small businesses nationwide – Generated >$57.8 billion in new economic activity— nearly double taxpayers’ investment

 NIH serves as foundation for entire U.S. medical innovation sector that:
– Has total employment impact of 7 million jobs – Exports $90 billion in goods, services
Profiles of Prosperity: How NIH-Supported Research Is Fueling Private Sector Growth and Innovation, United Medical Research, July 2013; An Economic Engine: NIH Research, Employment and the Future of the Medical Innovation Sector, United Medical Research, May 2011; State Bioscience Initiatives, BIO, June 2008

NIH Investments in Innovation
 Accelerating Discovery Through Technology  Advancing Translational Sciences  Encouraging New Investigators and New Ideas

5,671
$1,000,000 $100,000 $10,000

$100,000,000

$10,000,000

S-01 J-02 M-02 S-02 J-03 M-03 S-03 J-04 M-04 S-04 J-05 M-05 S-05 J-06 M-06 S-06 J-07 M-07 S-07 J-08 M-08 S-08 J-09 M-09 S-09 J-10 M-10 S-10 J-11 M-11 S-11 J-12 M-12 S-12 J-13

September 2001–January 2013

Cost of Sequencing a Human Genome

$1,000

The Cancer Genome Atlas
 Coordinated effort to accelerate understanding of cancer through genome analysis to improve ability to diagnose, treat, and prevent cancer  Provides analysis of > 20 types of cancer, including
– – – – Acute myeloid leukemia Breast Colon/rectal Endometrial (uterine) – Glioblastoma (brain) – Lung squamous cell carcinoma – Ovarian

The Cancer Genome Atlas: Comparing “Fingerprints” of Cancer Types
 Searching for shared genomic fingerprints
– 12 cancer types – Tumor samples from 3,300 patients

 Found 127 significantly mutated genes shared by subsets of samples across cancer types  Potential significance for …
– “Precision medicine” – customizing treatment for individual patients – Therapeutic development – identifying key cellular pathways to find likely drug targets

http://directorsblog.nih.gov/

ENCODE: Encyclopedia of DNA Elements
 Launched in 2003, to develop comprehensive catalog of functional elements in the human genome
– ENCODE consortium: including Anindya Dutta, UVA

 Community resource project
– All data free and available for immediate use

 Update 2012: 30+ papers ─ including main findings, innovative access, in Nature
– First global, detailed view of functional elements in human genome

www.encodeproject.org

“The Next Great American Project”

Learning the Language of the Brain

Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative
 Accelerate new technologies to
– Produce real-time pictures of complex neural circuits – Visualize rapid-fire interactions of cells at the speed of thought

 Open new doors to understanding
– How brain function is linked to human behavior and learning – Mechanisms of brain disease

NIH Investments in Innovation
 Accelerating Discovery Through Technology  Advancing Translational Sciences  Encouraging New Investigators and New Ideas

Disorders with Known Molecular Basis
5000 4500 4000 3500 3000 2500 2000 1500 1000 500 0

250 with therapy

Source: Online Mendelian Inheritance in Man, Morbid Anatomy of the Human Genome

Therapeutic Development Pipeline

6.5 years

6 years

1.5 years

National Center for Advancing Translational Sciences (NCATS)
Mission:
To advance the discipline of translational science and catalyze the development, testing, and implementation of novel diagnostics and therapeutics across a wide range of human diseases and conditions.

http://ncats.nih.gov/

Tissue Chip for Drug Screening:
Collaborating with Our Sister Agency in Virginia
 Goal: develop chip to screen for safe, effective drugs
– Liver, heart, lung, other cell types – Designed for multiple different readouts

 NIH, DARPA contribute ~$70M each over 5 years
– NIH/NCATS and DARPA independently manage, fund separate but highly coordinated program – FDA provides regulatory science guidance

 Awards announced in 2012
– Supporting the best ideas in engineering, biology, toxicology

Tissue Chips: Potential Uses
 Safety/toxicity: predict adverse events earlier to allow their prevention or mitigation  Efficacy: identify population that will respond to a new drug earlier to speed development  Efficiency: prevent missed opportunities with candidate drugs failing in early animal studies

Discovering New Therapeutic Uses for Existing Molecules
Partnership to match deprioritized compounds from pharma with innovative ideas for new indications
 Pharma partners: 58 compounds and biologics; associated data  NIH grantees: great ideas; patient access  NIH/NCATS: $12.7M/year to support staged, cooperative agreement research grants
– 9 awards issued in June 2013 in 8 disease areas • Virginia Commonwealth University: award to examine novel therapeutic to treat nicotine dependence

Therapeutics for Rare and Neglected Diseases (TRND) Program
 Encourages, speeds development of new drugs for rare and neglected diseases
– Collaborations between NIH and expert outside labs – Conducts pre-clinical development of new drugs; advances them to first-in-human clinical trials

 Example: Aes-103 to treat sickle cell disease
– Aes-103: drug candidate hinders sickling – discovered at Virginia Commonwealth University – Partnership: NIH and AesRx (biotech) – Phase IIa trial to start December 2013

Clinical and Translational Science Awards
A national consortium of more than 60 medical research institutions

Virginia Commonwealth University Center for Clinical and Translational Research
 Provides matrix to strengthen local ties – to improve response to community health needs  Focuses on the areas of strength:
– Substance abuse – Women’s health – Rehabilitation science

 Offers M.S. and Ph.D. degree programs in clinical and translational science  New partnership with Penn State’s CTSA will expand capacity, opportunities

NIH Investments in Innovation
 Accelerating Discovery Through Technology  Advancing Translational Sciences  Encouraging New Investigators and New Ideas

Nurturing Talent and Innovation
NIH initiatives to support a diverse, well-trained, highly creative workforce – essential to success of biomedicine:  Increasing diversity
– Building Infrastructure Leading to Diversity (BUILD) – National Research Mentoring Network (NRMN) – Broadening Experiences in Science Training (BEST)

 Supporting early-career investigators; cultivating innovation: NIH Director’s Awards
– – – – Early Independence Pioneer New Innovator Transformative Research

Nurturing Talent and Innovation in Virginia: National Research Mentoring Network (NRMN)
 NRMN goals:
– Develop network of skilled mentors linked to mentees across the country – Develop best practices for mentoring – Establish standards to assess efficacy

 New NRMN planning grants: Hampton University
– 6-month grant to develop multi-year plan – Hampton’s President William Harvey and team to devise plan for national mentoring consortium • Establish partnerships with array of institutions • Assess current mentoring strategies; enhance for national application • Delineate innovative mentoring methods
Dr. William R. Harvey Hampton University

Nurturing Talent and Innovation
NIH initiatives to support a diverse, well-trained, highly creative workforce – essential to success of biomedicine:  Increasing diversity
– Building Infrastructure Leading to Diversity (BUILD) – National Research Mentoring Network (NRMN) – Broadening Experiences in Science Training (BEST)

 Supporting early-career investigators; cultivating innovation: NIH Director’s Awards
– – – – Early Independence Pioneer New Innovator Transformative Research

Nurturing Talent and Innovation New Innovators in Virginia

Kevin A. Janes University of Virginia

Linsey Marr Virginia Tech

NIH Program Level
In Nominal Dollars and Constant 1998 Dollars
$35

$30

$25

$20

$15 (In Millions)

$10

$5

$0

Appropriation

Appropriation in 1998 Dollars

Success Rate
10% 15% 20% 25% 30% 35% 40% 45% 5%
1978 1979 1980 1981 1982 1983 1984 1985 1986 1987 1988 1989 1990 1991 1992 1993 1994 1995 1996 1997 1998 1999 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013

FY 1978-2013

Grant Success Rates

Fiscal Year

?

“If you're going through hell, keep going.”
– Winston Churchill

Anindya Dutta, M.D., Ph.D. Byrd Professor and Chair of Biochemistry and Molecular Genetics, UVA On Sequestration

"I am living off of fumes"

Our laws and institutions must go hand-in-hand with progress of the human mind.
~Thomas Jefferson

NIH…
Turning Discovery Into Health

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