NIH action plan charts future challenges for liver disease research


The National Institutes of Health (NIH) today released the trans-NIH Action Plan for Liver Disease Research, a comprehensive plan that addresses the burden of liver diseases in the United States and maps out challenges for future research. The Action Plan is available on-line at

"Over the last 25 years medical research in liver disease has greatly improved the survival and quality-of-life of patients with liver disease," said Allen M. Spiegel, M.D., Director of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the NIH institute with lead responsibility for drafting the plan. "This trans-NIH plan summarizes challenges to advancing liver disease research and delineates the major goals for future research."

Goals of the Action Plan for Liver Disease Research include:

  • Improving the success rate of therapy of hepatitis C. The currently recommended regimen of antiviral therapy results in long-term viral eradication in only 50-60 percent of patients; therefore, improved therapies are needed to benefit more patients with hepatitis C.

  • Developing noninvasive ways to measure liver fibrosis. Currently, liver biopsy is the standard means of evaluating the severity of liver disease both in clinical practice as well as in clinical trials of new therapies. Availability of reliable and safe means of measuring fibrosis (tissue scarring) and cirrhosis (excessive scarring and destruction of liver structure) would improve management of liver disease and facilitate clinical research.

  • Developing sensitive and specific means of screening individuals at high risk for early hepatocellular carcinoma (liver cancer). Liver cancer is often fatal and difficult to diagnose at an early stage when it is easier to treat and possible to cure by surgery. Simple and accurate diagnostic techniques could detect liver cancer early and improve patient survival.

  • Developing standardized and objective diagnostic criteria of major liver diseases and their grading and staging. Standards will benefit clinical research on all types of liver disease, including the evaluation of new diagnostic and therapeutic agents.

  • Decreasing the mortality rate from liver disease. Improved means of prevention and treatment should enable a decrease of at least 20 percent in the age-adjusted death rates from liver disease in the United States.

    Liver disease is an important cause of sickness and death in the United States. According to statistics from the Centers for Disease Control and Prevention (CDC), liver and biliary (gallbladder) disease, including liver cancer, accounts for about 46,000 deaths each year and ranks ninth in overall causes of death. Because of the high rates of liver disease, liver transplantation is now considered standard therapy for patients with end-stage liver disease, regardless of diagnosis. Currently, about 5,000 liver transplants are performed yearly in the United States at more than 120 medical centers. As a consequence of the limited supply of livers, there are more than 17,000 persons on the liver transplant waiting list and at least 1,500 will die annually while waiting.

    "Acute and chronic liver disease affects people of all ages, with the greatest burden among minority individuals and persons between the ages of 40 and 60," said Jay H. Hoofnagle, M.D., director, NIDDK's Liver Disease Research Branch. "The major focus of this Action Plan is to stimulate translation of basic research findings to practical and effective means of prevention and control of liver diseases, including such important conditions as hepatitis B and C, biliary atresia, liver cancer, alcoholic and nonalcoholic fatty liver, primary biliary cirrhosis, and autoimmune hepatitis. The explosion of knowledge about fundamental biology and genetics in the past 20 years now promises to provide significant improvements in management of liver disease and specific prevention of many of these important causes of disease and mortality among Americans."

    In 2003, the NIH created the Liver Disease Research Branch to focus and accelerate research on liver disease at the NIDDK and to coordinate liver-related research across the NIH and among other federal agencies. The Branch worked with the Liver Disease Subcommittee of the congressionally authorized Digestive Diseases Interagency Coordinating Committee to coordinate the drafting of the Action Plan. Representatives from 17 NIH institutes and approximately 250 experts in liver disease research from across the country, including researchers, practicing physicians, academicians, representatives of liver disease professional societies, patient voluntary and non-profit groups, and other interested members of the public, participated actively in the development of the plan.

    "This year-long process was open and inclusive and engaged a great number of scientists and experts in the field of liver disease research," said Hoofnagle. "We have highlighted specific goals for the next ten years that are focused upon further reducing the frequency and burden of liver disease."

    Along with introductory and summary statements, each chapter of the Action Plan includes a background section, a summary of recent advances, a central section describing important future research goals, and a final section that outlines active steps to achieve each goal. A total of 214 research goals identified in the Action Plan are categorized by their degree of difficulty and time frame, with several research goals overlapping. The Action Plan also includes an implementation plan and a list of benchmark goals that will be used to gauge its effectiveness and success.

    Source: Eurekalert & others

    Last reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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