The Role of the NIH Clinical Center in Translational and Clinical Research

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Bethany Glenn
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1 The Role of the NIH Clinical Center in Translational and Clinical Research International Conference on Engineering Science and Technology 2014 John I. Gallin, M.D. Director, NIH Clinical Center Associate Director for Clinical Research June 2, 2014

2 Outline NIH Overview and Current Areas of Emphasis in Clinical Research New Activities at the NIH Clinical Center

The National Institutes of Health The Nation

systems and the application of that knowledge

3 The National Institutes of Health The Nation s Steward of Medical & Behavioral Research 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.

4 The National Institutes of Health NCI NINR NCCAM NHLBI OD NIAMS NIEHS NIMHD NIDA NLM NIMH NEI NHGRI NIBIB NCATS NINDS NIDCR NIDDK NIGMS FIC NIA NIAAA NIAID NICHD NIDCD CSR CC CIT Extramural only No Funding Authority

5 NIH Extramural & Intramural Funding FY 2014: $30.15 Billion Spending at NIH Spending Outside NIH

6 NIH s Commitment to Basic Research Applied Research Basic Research

7 NIH s Impact on U.S. Health and Medicine 80 U.S. Life Expectancy Life expectancy gains worth ~$3.2 trillion annually NIH Accomplishments Reduction Cardiovascular in deaths disease from: Heart death disease rates have fallen > 60% Stroke in the last half-century HIV HIV/AIDS therapies enable people in their 20s to live to age 70+ Increased survival rates for: Cancer Breast cancer rates now falling ~1% Cervical per year; cancer each 1% drop Colon saves cancer ~$500 billion

8 Biomedical Research Stimulates the Economy In 2011 NIH funding generated $62 billion in new U.S. economic activity double the taxpayers investment

9 NIH Leading the Way Genomics Cellular Therapy/Regenerative Medicine Vaccine Development Human Microbiome BRAIN initiative Big Data Problem

10 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 M-13 S-13 J-14 Cost of Sequencing a Human Genome September 2001 January 2014 $100,000,000 $10,000,000 $1,000,000 $100,000 $10,000 4,008 $1,000

1987 1988 1989 1990 1991 1992 1993 1994 1995 1996 1997

2009 2010 2011 2012 2013 2014 Disorders with Known

2000 1500 1000 500 0 ~500 with therapy Source: Online

11 Disorders with Known Molecular Basis ~500 with therapy Source: Online Mendelian Inheritance in Man, Morbid Anatomy of the Human Genome

The Cancer Genome Atlas: Comparing Fingerprints of Cancer Types Coordinated effort to accelerate

cancer Searching for shared genomic fingerprints 12 cancer types Tumor samples from 3,300 patients

significance for Therapeutic development identifying key cellular pathways to find likely drug

12 The Cancer Genome Atlas: Comparing Fingerprints of Cancer Types Coordinated effort to accelerate understanding of cancer through genome analysis to improve ability to diagnose, treat, and prevent cancer 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 Therapeutic development identifying key cellular pathways to find likely drug targets Precision medicine customizing treatment for individual patients

13 Developing Promise of Cellular Therapy ipscs To study toxicology Generate tissues and cells for transplantation Understand how to prevent and treat birth defects Cellular therapy

14 Vaccine Development The Need for a Universal Flu Vaccine Up to 50,000 U.S. deaths associated with flu annually $87 billion in economic costs Protection currently involves getting a flu shot every year Universal vaccine: one shot provides lasting immunity to most strains Vaccine would also protect against an emerging flu pandemic

15 The Human Microbiome: Transforming Our Understanding of Health and Disease Herbivores Omnivores Carnivores

16 The Brain Initiative The Next Great American Project Learning the Language of the Brain (NIH initial investment $40M)

BRAIN Initiative NIH goals Generate a Census of Cell

Large-Scale Network Recording Capabilities Develop A

Activity to Behavior Integrate Theory, Modeling,

17 BRAIN Initiative NIH goals Generate a Census of Cell Types Create Structural Maps of the Brain Develop New Large-Scale Network Recording Capabilities Develop A Suite of Tools for Circuit Manipulation Link Neuronal Activity to Behavior Integrate Theory, Modeling, Statistics, and Computation with Experimentation Assure ethical issues considered

18 The Big Data Problem

19 Exposure Clinical Myriad Data Types Genomic Other Omic Imaging Phenotypic

20 NIH Is Tackling the Big Data Problem Big Data to Knowledge (BD2K) Major trans-nih initiative Goal: by decade's end, enable a quantum leap in the ability of the biomedical research enterprise to maximize the value of the growing volume and complexity of biomedical data

21 Despite advances and promise, translational research has problems Slow Expensive NIH National Center for Advancing Translational Sciences

22 NIH Clinical Center World s largest clinical research center Founded 1953 There s no other hospital like it

23 Clinical Center Profile >480,000 patients since opening in beds; 2014 Budget $402.8M Hospital surrounded by research labs Every patient is on a research protocol Care is free Patient travel/housing provided as needed ~6,500 employees 1,255 credentialed physicians 1,530 active protocols Interventional/Clinical Trials (47%) Natural History (47%) Screening - 66 (4%) Training - 26 (2%)

24 Major Emphasis Study of the pathophysiology of disease First in human with new therapeutics Study of patients with rare diseases million people in the United States have a rare disease!

25 Rare Diseases: Window on Nature? Nature is nowhere accustomed more openly to display her secret mysteries than in cases where she shows traces of her workings apart from the beaten path; nor is there any better way to advance the proper practice of medicine than to give our minds to the discovery of the usual law of nature, by the careful investigation of cases of rarer forms of disease. ~ William Harvey, Letter IX, to John Vlackveld, 24 Apr 1657

Disease ( HDL) Immunoregulation (Janus Kinases) Inhibitors (under development) Statins Tofacitinib for RA,

26 Studies at CC of Rare Diseases with Impact X-SCIDS on a Common Disease Disease Model for Drug Choriocarcinoma Cancer Farnesyltransferase Chemotherapy Progeria Tangier Lamin A in Aging Cardiovascular Disease ( HDL) Immunoregulation (Janus Kinases) Inhibitors (under development) Statins Tofacitinib for RA, Psoriasis, Ulcer.colitis Photo: The Progeria Research Foundation Source: NIH Director s Blog

Clinical Center-The Future Disease prevention Linkage of genotype and phenotype to develop new therapeutics for preventing and treating disease Cellular therapy Imaging For immunotherapy of certain

27 Clinical Center-The Future Disease prevention Linkage of genotype and phenotype to develop new therapeutics for preventing and treating disease Cellular therapy Imaging For immunotherapy of certain cancers and for organ replacement Detection of finer and finer perturbations of structure and function to diagnose and stage disease Information technology Develop portals to merge diverse data sets to guide research and future patient care Training tomorrow s translational and clinical investigators

28 New NIH Grants Opportunities for Collaborative Research at the NIH Clinical Center Supports collaborative research to enhance the translation of basic biological discoveries into clinical applications that improve health Up to $500K/year in direct costs x 3 years, renewable Teams must have 1 outside and 1 intramural co-principal investigator; international partners welcome Some work must be done at the Clinical Center resources/

29 Concluding Messages Solving health issues is a global challenge requiring international cooperation. The NIH Clinical Center is a special resource and by opening its doors to investigators in the United States and internationally we hope to continue to catalyze future research.

30 Thank you!

NIH Institutes and Areas of SBIR Research: (shown in order of FY 2010 Funding amounts)

NIH Institutes and Areas of SBIR Research: (shown in order of FY 2010 Funding amounts) 1. NCI (National Cancer Institute) cancer cause, prevention, detection, diagnosis, treatment and control 2. NIAID