| 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 http://liverplan.niddk.nih.gov.
“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
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
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.