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Biennial Report of the Director

Research in Diseases, Disorders, and Health Conditions
Chronic Diseases and Organ Systems
Digestive Diseases

Digestive diseases span a wide spectrum of diseases and disorders that affect the gastrointestinal (GI) tract, liver, gallbladder, and pancreas, many forms of which are chronic. Some digestive diseases are common, such as gastroesophageal reflux disease (GERD), while others are quite rare, such as genetic forms of liver disease. However, collectively they exact a significant toll on public health in terms of quality of life, years of life lost due to premature death, and costs associated with hospitalizations and pharmaceutical and surgical interventions. Additional information on some chronic digestive conditions and diseases is included in other sections of this report (e.g., irritable bowel syndrome under Chronic Pelvic Pain, inflammatory bowel diseases and celiac disease under Autoimmune Diseases, cancers of digestive system under Cancer, and viral hepatitis under Infectious Diseases and Biodefense).

To reduce the public health burden of digestive diseases, NIH-supported scientists are vigorously pursuing research to better understand how widespread these diseases are across the U.S. and in specific population groups, to identify the causes of diseases and how they progress, and to test new interventions for prevention and treatment of these costly diseases, including drugs, surgery, and behavior modification. Major supporters of digestive diseases research at NIH include NIDDK, as well as NCI and NIAID.

In addition to its extensive portfolio supporting individual investigators advancing digestive diseases research, NIDDK also supports multi-center research efforts such as the Inflammatory Bowel Disease Genetics Consortium, Gastroparesis Clinical Research Consortium, Nonalcoholic Steatohepatitis Clinical Research Network, Childhood Liver Disease Research Network, Drug-Induced Liver Injury Network, adult and pediatric Acute Liver Failure Study Groups, and Hepatitis B Research Network, as well as 21 Digestive Diseases Research Centers located across the country. The Institute also supports follow-up and ancillary studies to completed clinical trials in such areas as hepatitis C and adult-to-adult living donor liver transplantation.
Recent advances promise new approaches for treating and preventing these pervasive and debilitating disorders. A group of NIDDK-sponsored scientists has developed a method for turning human adult stem cells that are pluripotent (i.e., have the capability becoming different tissue cell type), into three-dimensional intestinal tissue in culture. This pioneering work has the potential to open up several new research directions, including elucidating pathways involved in inherited intestinal conditions, testing new drugs for their intestinal absorption, and even generating tissue for transplantation in conditions such as inflammatory bowel diseases, necrotizing enterocolitis, and short-gut syndromes.159

Researchers in NIDDK’s Nonalcoholic Steatohepatitis Clinical Research Network have shown benefits of vitamin E as a treatment for nonalcoholic steatohepatitis (fatty liver disease) in adults, as well as in children with the most severe form of the disease. Nonalcoholic fatty liver disease is the most common chronic liver disease among U.S. children.160 In other advances, NIDDK-sponsored investigators successfully implanted physiologically functional, bioengineered anal sphincters in mice, which could lead to the development of a treatment for fecal incontinence in humans.161 An NCCAM-supported trial showed that placebos administered with the patients’ knowledge may be an effective treatment for IBS.162 Researchers in the NIDDK Gastroparesis Clinical Research Consortium discovered evidence of changes at the cellular level in the stomachs of individuals with gastroparesis, a chronic condition characterized by impaired gastrointestinal motility, yielding new insights into this digestive disorder.163

NIH, along with Federal and non-Federal partners, will continue efforts to address goals for advancing digestive diseases research in the NIH-led National Commission on Digestive Diseases research plan.164 For example, the NIDDK, together with NIAID, has established a new Intestinal Stem Cell Consortium, which is based on a research recommendation from the National Commission on Digestive Diseases’ research plan, to stimulate basic research on the digestive system by developing new technologies to isolate, characterize, cultivate, and manipulate its stem cells.

Additionally, NIDDK is continuing support of its current research efforts in digestive diseases, as well as pursuing new directions. For example, NIDDK is supporting new studies through the Hepatitis B Research Network to advance understanding of disease processes and to develop effective approaches to treatment. In 2012, the NIDDK entered into a cooperative research and development agreement (CRADA) with a pharmaceutical company to begin a clinical trial testing the safety and potential efficacy of a new treatment—cysteamine bitartrate—for nonalcoholic steatohepatitis (NASH) in children through the NASH Clinical Research Network.164 The NIDDK also launched a Bowel Control Awareness Campaign to help patient and healthcare professionals feel more comfortable talking about conditions such as fecal incontinence.165


158 Spence JR, et al. Nature. 2010;470:105–9. PMID: 21151107.
159 Sanyal AJ, et al. NEJM. 2010;362(18):1675–85. PMID: 20427778; Lavine JE, et al. JAMA. 2011;305(16):1659–68. PMID: 21521847.
160 Raghavan S, et al. Am J Physiol Gastrointest Liver Physiol. 2010;299(2):G430–9. PMID: 20558766; Hashish M, et al. J Pediatr Surg. 2010;45(1):52–8. PMID: 20105579.
161 Kaptchuk TJ, et al. PLoS ONE. 2010;5(12):e15591. PMID: 21203519.
162 Grover M, et al. Gastroenterology. 2011;140(5):1575–1585. PMID: 21300066.
163 For more information, see: https://www2.niddk.nih.gov/AboutNIDDK/CommitteesAndWorkingGroups/NCDD/FinalResearchPlanPosting.htm.
164 For more information, see https://www.nih.gov/news/health/jan2012/niddk-11.htm.
165 For more information, see https://www.bowelcontrol.nih.gov/.