Progress in Autoimmune Diseases Research

Progress in

Autoimmune Diseases Research

National Institutes of Health

T H E A U T O I M M U N E D I S E A S E S C O O R D I N AT I N G C O M M I T T E E

Report to Congress

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES

Progress in

Autoimmune Diseases Research

National Institutes of Health

T H E A U T O I M M U N E D I S E A S E S C O O R D I N AT I N G C O M M I T T E E

U.S. DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health

National Institute of Allergy and Infectious Diseases

March 2005

Foreword

A

utoimmune diseases are a family of more than 80 chronic, and often disabling,

illnesses that develop when underlying defects in the immune system lead the

body to attack its own organs, tissues, and cells. While many of these dis?

eases are rare, collectively they affect 14.7 to 23.5 million people in this country, and ¨C for

reasons unknown ¨C their prevalence is rising. Since cures are not yet available for most

autoimmune diseases, patients face a lifetime of illness and treatment. They often endure

debilitating symptoms, loss of organ function, reduced productivity at work, and high medical

expenses. And, because most of these diseases disproportionately afflict women, and are

among the leading causes of death for young and middle-aged women, they impose a

heavy burden on patients¡¯ families and on society.

For these reasons, Congress commissioned the Autoimmune Diseases Coordinating

Committee (ADCC) to develop a comprehensive strategic Research Plan for National

Institutes of Health (NIH)-funded autoimmune research with the goal of reducing the

impact of autoimmune disease. Published in January 2002, the Autoimmune Diseases

Research Plan was developed by ADCC members, other Federal and non-Federal experts

in the field, and lay leaders in the autoimmune disease communities. It sets forth an ambitious

and comprehensive research agenda aimed at generating more accurate epidemiologic

profiles of autoimmune diseases; developing a greater understanding of the fundamental

biologic principles underlying disease onset and progression; devising improved

diagnostic tools; creating more effective interventions; and producing public and professional

education and training programs.

Although it has been a short time since the Research Plan was established, we have seen

significant progress in all of these areas ¨C progress that has resulted from the combined

efforts of NIH Institutes and Centers, other Federal agencies, nonprofit and patient advo?

cacy groups, and partners in the private sector. This report details the progress made in

NIH-funded autoimmune research since publication of the 2002 ADCC Autoimmune

Diseases Research Plan.

It also identifies overarching priority areas that promise to accelerate autoimmune disease

research. These areas include biomarker development, bioinformatics, and application of

new technologies. The development of biomarkers can enable earlier diagnosis as well as

aid physicians in selecting and monitoring treatment. New technologies, such as genomics

and proteomics, provide scientists with the tools to study gene and protein patterns in

tissue samples, providing vital insights into the onset and progression of disease.

Bioinformatics tools, which help scientists to assemble and analyze large amounts of data,

will be particularly important in these endeavors.

In many of these areas, the NIH

Roadmap, which fosters trans-Institute and multidisciplinary collaboration as a way to

address complex challenges in biomedical research, will synergize with the ADCC

Autoimmune Diseases Research Plan.

Despite our progress, we recognize that more needs to be done so that we may close

the gaps in our knowledge and achieve our overall goal of reducing the rising toll of

autoimmune disease. For example, we need to gain a better understanding of the distri?

bution of these diseases through epidemiologic studies, and of the environmental triggers

that contribute to their onset. We must work to apply the knowledge provided by the

Human Genome Project toward elucidating the hereditary risks of autoimmune diseases.

As we learn more about the genetic and environmental factors contributing to

these diseases, we will be able to develop effective prevention strategies that arrest the

autoimmune process before it can irreversibly damage the body. In tandem, we must

advance the training of scientists and health care workers so that we can effectively trans?

late the advances in biomedical research to clinical practice.

NIH is deeply committed to supporting research and promoting progress toward con?

quering autoimmune diseases. The 2002 ADCC Autoimmune Diseases Research Plan

provides a valuable guide to those goals. Reporting on our advances, as we do in this

Progress Report, offers an opportunity to evaluate our progress, assess our path, and

determine our direction for the future. Ultimately, basic and clinical research in this area will

yield more effective prevention and treatment strategies for the millions who are at risk for

or struggle with autoimmune diseases.

Elias A. Zerhouni, M.D.

Director

National Institutes of Health

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