Table of Contents What Is Scleroderma? What Are the Different Types of Scleroderma? What Causes Scleroderma? Who Gets Scleroderma? How Can Scleroderma Affect My Life? Symptoms of Scleroderma What Other Conditions Can Look Like Scleroderma? Scleroderma Treatment How Can I Play a Role in My Health Care? Scleroderma Research More Questions? Keeping on Top of Your Condition

Scleroderma Research

No one can say for sure when--or if--a cure will be found. But research is providing the next best thing: better ways to treat symptoms, prevent organ damage, and improve the quality of life for people with scleroderma. In the past two decades, multidisciplinary research has also provided new clues to understanding the disease, which is an important step toward prevention or cure.

Leading the way in funding for this research is the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a part of the National Institutes of Health (NIH). Other sources of funding for scleroderma research include pharmaceutical companies and organizations such as the Scleroderma Foundation, the Scleroderma Research Foundation, and the Arthritis Foundation. Scientists at universities and medical centers throughout the United States conduct much of this research.

Studies of the immune system, genetics, cell biology, and molecular biology have helped reveal the causes of scleroderma, improve existing treatment, and create entirely new treatment approaches.

Research advances in recent years that have led to a better understanding of and/or treatment for the diseases include:

• The use of a hormone produced in pregnancy to soften skin lesions. Early studies suggest relaxin, a hormone that helps a woman's body to stretch to meet the demands of a growing pregnancy and delivery, may soften the connective tissues of women with scleroderma. The hormone is believed to work by blocking fibrosis, or the development of fibrous tissue between the body's cells.
  
  
• Finding a gene associated with scleroderma in Oklahoma Choctaw Native Americans. Scientists believe the gene, which codes for a protein called fibrillin-1, may put people at risk for the disease.
  
  
• The use of the drug Iloprost for pulmonary hypertension. This drug has increased the quality of life and life expectancy for people with this dangerous form of lung damage.
  
  
• The use of the drug cyclophosphamide (Cytoxan) for lung fibrosis. One recent study suggested that treating lung problems early with this immunosuppressive drug may help prevent further damage and increase chances of survival.
  
  
• The increased use of ACE inhibitors for scleroderma-related kidney problems. For the past two decades, ACE inhibitors have greatly reduced the risk of kidney failure in people with scleroderma. Now there is evidence that use of ACE inhibitors can actually heal the kidneys of people on dialysis for scleroderma-related kidney failure. As many as half of people who continue ACE inhibitors while on dialysis may be able to go off dialysis in 12 to 18 months.
  

Other studies are examining the following:

• Changes in the tiny blood vessels of people with scleroderma. By studying these changes, scientists hope to find the cause of cold sensitivity in Raynaud's phenomenon and how to control the problem.
  
  
• Immune system changes (and particularly how those changes affect the lungs) in people with early diffuse systemic sclerosis.
  
  
• The role of blood vessel malfunction, cell death, and autoimmunity in scleroderma.
  
  
• Skin changes in laboratory mice in which a genetic defect prevents the breakdown of collagen, leading to thick skin and patchy hair loss. Scientists hope that by studying these mice, they can answer many questions about skin changes in scleroderma.
  
  
• The effectiveness of various treatments, including (1) methotrexate, a drug commonly used for rheumatoid arthritis and some other inflammatory forms of arthritis; (2) collagen peptides administered orally; (3) halofugione, a drug that inhibits the synthesis of type I collagen, which is the primary component of connective tissue; (4) ultraviolet light therapy for localized forms of scleroderma; and (5) stem cell transfusions, a form of bone marrow transplant that uses a patient's own cells, for early diffuse systemic sclerosis.

Scleroderma research continues to advance as scientists and doctors learn more about how the disease develops and its underlying mechanisms.

Recently, the NIAMS funded a Specialized Center of Research (SCOR) in scleroderma at the University of Texas-Houston. SCOR scientists are conducting laboratory and clinical research on the disease. The SCOR approach allows researchers to translate basic science findings quickly into improved treatment and patient care.