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Precision Medicine in Pancreatic Disease—Knowledge Gaps and Research Opportunities

Summary of a National Institute of Diabetes and Digestive and Kidney Diseases Workshop

Lowe, Mark E. MD, PhD*; Andersen, Dana K. MD; Caprioli, Richard M. PhD‡,§,∥; Choudhary, Jyoti PhD; Cruz-Monserrate, Zobeida PhD#; Dasyam, Anil K. MD**; Forsmark, Christopher E. MD††; Gorelick, Fred S. MD‡‡,§§,∥∥; Gray, Joe W. PhD¶¶; Haupt, Mark MD##; Kelly, Kimberly A. PhD***; Olive, Kenneth P. PhD†††,‡‡‡; Plevritis, Sylvia K. PhD§§§,∥∥∥; Rappaport, Noa PhD¶¶¶; Roth, Holger R. PhD###; Steen, Hanno PhD****; Swamidass, S. Joshua MD, PhD††††; Tirkes, Temel MD‡‡‡‡; Uc, Aliye MD§§§§; Veselkov, Kirill PhD∥∥∥∥; Whitcomb, David C. MD, PhD¶¶¶¶,####,*****; Habtezion, Aida MD, MSc†††††

doi: 10.1097/MPA.0000000000001412
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A workshop on research gaps and opportunities for Precision Medicine in Pancreatic Disease was sponsored by the National Institute of Diabetes and Digestive Kidney Diseases on July 24, 2019, in Pittsburgh. The workshop included an overview lecture on precision medicine in cancer and 4 sessions: (1) general considerations for the application of bioinformatics and artificial intelligence; (2) omics, the combination of risk factors and biomarkers; (3) precision imaging; and (4) gaps, barriers, and needs to move from precision to personalized medicine for pancreatic disease. Current precision medicine approaches and tools were reviewed, and participants identified knowledge gaps and research needs that hinder bringing precision medicine to pancreatic diseases. Most critical were (a) multicenter efforts to collect large-scale patient data sets from multiple data streams in the context of environmental and social factors; (b) new information systems that can collect, annotate, and quantify data to inform disease mechanisms; (c) novel prospective clinical trial designs to test and improve therapies; and (d) a framework for measuring and assessing the value of proposed approaches to the health care system. With these advances, precision medicine can identify patients early in the course of their pancreatic disease and prevent progression to chronic or fatal illness.

From the *Department of Pediatrics, School of Medicine, Washington University, St Louis, MO

The Division of Digestive Diseases and Nutrition, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD

Departments of Biochemistry

§Chemistry

Pharmacology and Medicine, Vanderbilt University, Nashville, TN

Institute of Cancer Research, London, United Kingdom

#Division of Gastroenterology, Hepatology, and Nutrition, Comprehensive Cancer Center, The Ohio State University Wexner Medical Center, Columbus, OH

**Department of Radiology, University of Pittsburgh, Pittsburgh, PA

††The Division of Gastroenterology, Hepatology and Nutrition, University of Florida, Gainesville, FL

Departments of ‡‡Internal Medicine

§§Cell Biology, School of Medicine, Yale University, New Haven, CT

∥∥VA Connecticut Healthcare System, West Haven, CT

¶¶Knight Cancer Institute, Oregon Health and Science University, Portland, OR

##Ariel Precision Medicine, Pittsburgh, PA

***Department of Biomedical Engineering, University of Virginia, Charlottesville, VA

†††Department of Medicine

‡‡‡Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY

Departments of §§§Biomedical Data Science

∥∥∥Radiology, Stanford University, Stanford, CA

¶¶¶Institute for Systems Biology, Seattle, WA

###Deep Learning for Medical Imaging, NVIDIA Corporation, Bethesda, MD

****Proteomics Center, Boston Children's Hospital, Department of Pathology, Harvard Medical School, Boston, MA

††††Department of Pathology and Immunology, School of Medicine, Washington University, St Louis, MO

‡‡‡‡Department of Radiology and Imaging Sciences, School of Medicine, Indiana University, Indianapolis, IN

§§§§Division of Gastroenterology, Stead Family Department of Pediatrics, The University of Iowa, Iowa City, IA

∥∥∥∥Computational Medicine and Systems Medicine, Department of Digestion, Metabolism and Reproduction, Imperial College, London, United Kingdom

Departments of ¶¶¶¶Medicine

####Cell Biology

*****Physiology and Genetics, University of Pittsburgh, Pittsburgh, PA

†††††Division of Gastroenterology and Hepatology, Stanford University, Stanford, CA.

Received for publication August 5, 2019; accepted August 8, 2019.

Address correspondence to: Aida Habtezion, MD, MSc, Division of Gastroenterology and Hepatology, Stanford University, 300 Pasteur Dr, Room M211, Stanford, CA 94305 (e-mail: aida.habtezion@stanford.edu); or Mark E. Lowe, MD, PhD, Department of Pediatrics, School of Medicine, Washington University, 660 S Euclid Ave, MPRB 4th Floor, Campus Box 8208, St Louis, MO 63110 (e-mail: lowe@wustl.edu).

This study was supported by the National Cancer Institute and National Institute of Diabetes and Digestive and Kidney Diseases under the following award numbers: U01DK108300 (A.H.), U01DK108306 (D.C.W.), UO1DK108300 (C.E.F.), U01DK108323 (T.T.), and U01DK108334 (A.U., M.E.L.). M.E.L., D.K.A., R.M.C., Z.C-M., C.E.F., F.S.G., J.W.G., K.A.K., S.K.P., H.S., S.J.S., T.T., A.U., D.C.W., and A.H. reported funding from the National Institutes of Health; M.E.L., Z.C-M., C.E.F., H.S., T.T., A.U., D.C.W., and A.H. acknowledge the support of The Consortium for the Study of Chronic Pancreatitis, Diabetes and Pancreatic Cancer (CPDPC), funded by the National Cancer Institute and the National Institute of Diabetes and Digestive and Kidney Diseases; received research support from NIHR Biomedical Research Center of Imperial College of London (V.A.K.), Research Councils of UK (J.C.), Veterans Administration (F.S.G.), Vodafone Foundation (V.A.K.), Waters Corporation (V.A.K.), Wellcome Trust (J.C.). The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

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