Scleroderma research receives a boost from multiple NIH grants

February 22, 2002

Ten new research grants on scleroderma (systemic sclerosis) have been funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) at the National Institutes of Health (NIH). The grants, totaling more than $2 million per year, include both basic and clinical research studies. The Office of Research on Women's Health (ORWH) co-funded two of the grants.

"These research grants will provide new clues to understanding this disease, which is an important step towards prevention and cure," said Stephen I. Katz, M.D., Ph.D., director of the NIAMS. "The grants complement our already considerable investment in scleroderma research, which includes support of two specialized centers of research on the disease, as well as the recent funding of a Multidisciplinary Clinical Research Center on pediatric rheumatic diseases, such as juvenile scleroderma."

Scleroderma, often referred to as a single disease, is actually a symptom of a group of diseases that involves the abnormal growth of connective tissue, which supports the skin and internal organs. In some forms of scleroderma, hard, tight skin is the extent of the disease. In other forms, however, the problem goes much deeper, affecting blood vessels and internal organs, such as heart, lungs, and kidneys. In scleroderma, the immune system is thought to stimulate cells called fibroblasts to produce excess collagen.

Currently, there is no treatment that controls or stops the underlying problem: the overproduction of collagen. Little is known about the cellular changes that cause the skin and organs to harden, and the disease may be difficult to diagnose. Understanding the early cellular and molecular changes in scleroderma will help scientists to develop more effective treatments. The following projects will increase our understanding of the causes of scleroderma and bring us closer to finding treatments:

Cellular and Molecular Processes in Scleroderma

Immune Recognition of Modified Antigen in Scleroderma, Robert W. Hoffman, M.D., University of Missouri, Columbia. This study investigates the biochemistry of a characteristic autoantibody seen in scleroderma.

The Molecular Basis for Endothelial Dysfunction in Systemic Sclerosis, Bashar Kahaleh, M.D., Medical College of Ohio at Toledo. Small blood vessel abnormalities are seen in early scleroderma. This project investigates molecular and cellular mechanisms that may cause this dysfunction.

Fine Specificity of Scleroderma Autoantibodies, Judith A. James, M.D., Oklahoma Medical Research Foundation, Oklahoma City. This study examines the autoantibodies and where they bind in the tissue to determine their role in the development of scleroderma. (Co-funded by ORWH)

Study of Persistent Infection in Systemic Sclerosis Skin and Vessels, Maureen D. Mayes, M.D., Wayne State University, Detroit. This project examines persistent bacterial infection of the skin or small blood vessels as a potential cause of scleroderma. Results could lead to treatments that target the bacterial infection.

Studies of Collagen Gene Regulation in Two Murine Models of Scleroderma, Stephen H. Clark, Ph.D., University of Connecticut School of Medicine and Dentistry, Farmington. This project uses two mouse models to better understand the molecular mechanisms that increase accumulation of collagen in the skin characteristic of scleroderma. (Co-funded by ORWH)

Self- and Foreign-Lipids Presented by CD1 in Scleroderma, Michael S. Vincent, M.D., Brigham and Women's Hospital, Boston. This study investigates the interactions of CD1 proteins and T cells in the development of scleroderma.

Cell Transfer Between Mother and Child and Scleroderma HLA Alleles, Self-Peptides and Microbial Mimicry in Systemic Sclerosis, J. Lee Nelson, M.D., Fred Hutchinson Cancer Research Center, Seattle. Blood cells move between mother and child during pregnancy and may remain in either or both. This project studies blood cells from scleroderma patients and controls to determine their origin and role in the development of scleroderma.

T Cells in the Pathogenesis of Systemic Sclerosis, Chris D. Platsoucas, Ph.D., Temple University, Philadelphia. Later in life, maternal immune cells can be found in the child and fetal immune cells in the mother. This study will investigate the origin and role of these immune cells in the skin of scleroderma patients.

Research Projects on Innovative Therapies for Scleroderma:

Chemokine Antagonists in a Murine Model for Scleroderma, Anita C. Gilliam, M.D., Case Western Reserve University, Cleveland. This project uses a mouse model to study the early inflammatory events of skin fibrosis. This research will facilitate the early diagnosis of scleroderma so that treatment can be more effective. The mouse model allows investigators to test inhibitors to inflammation in animals before testing them in humans.

UV-Induced Collagen Reduction for Treating Skin Scleroderma, Sewon Kang, M.D., University of Michigan at Ann Arbor. This study tests the effectiveness of UV phototherapy for the treatment of localized forms of scleroderma.

The award of these grants is the result of the special solicitation for research applications on scleroderma, AR-00-007 entitled "Molecular Pathogenesis and New Interventions in Scleroderma"
( This RFA was based in part on the scientific opportunities identified in the conference cosponsored by NIAMS, "Emerging Opportunities in Scleroderma Research." A summary of the conference can be found at

The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) is to support research into the causes, treatment, and prevention of arthritis and musculoskeletal and skin diseases, the training of basic and clinical scientists to carry out this research, and the dissemination of information on research progress in these diseases. For more information about NIAMS, call the information clearinghouse at (301) 495-4484 or (877) 22-NIAMS (free call) or visit the NIAMS Web site at

The Office of Research on Women's Health (ORWH) was established in September 1990 within the Office of the Director, NIH. ORWH works in partnership with the NIH institutes and centers to ensure that women's health research is part of the scientific framework at NIH and throughout the scientific community. For more information about ORWH, call (301) 402-1770 or visit the ORWH Web site at

For more information on scleroderma, contact the following organizations:
American Academy of Dermatology
930 N. Meacham Road
P.O. Box 4014
Schaumburg, IL 60168-4014
Tel: (847) 330-0230 or toll free: (888) 462-3376
Fax: (847) 330-0050

American College of Rheumatology
1800 Century Place, Suite 250
Atlanta, GA 30345
Tel: (404) 633-3777
Fax: (404) 633-1870

Scleroderma Foundation
12 Kent Way, #101
Byfield, MA 01922
Tel: (800) 722-HOPE (free of charge) or (978) 463-5843
Fax: (978) 463-5809

Scleroderma Research Foundation
2320 Bath Street, Suite 315
Santa Barbara, CA 93105
Tel: (800) 441-CURE (2873) (free of charge) or (805) 563-9133
Fax: (805) 563-2402

Arthritis Foundation
1330 West Peachtree Street
Atlanta, GA 30309
Tel: (404) 872-7100 or (800) 283-7800 (free of charge)
Fax: (404) 872-0457

NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases

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