No one can say for sure when a cure will be found. Research, however, 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 for understanding the disease, which is an important step toward prevention and cure.  Scientists at universities and medical centers throughout Canada, the United States and other parts of the world are actively conducting research in the area of scleroderma. 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.

Some advances in the understanding or treatment of scleroderma include the following:

• Building on research that identified a gene associated with scleroderma in Oklahoma Choctaw Native Americans, scientists are using new technology to look for other genes associated with the disease’s development and severity.
• The drug cyclophosphamide has been found effective in treating 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. In further research assessing the impact of cyclophosphamide on quality of life in people with lung involvement, 47 percent of people on the drug reported their health was somewhat or much better after 1 year, compared with 18 percent in the placebo group.
• ACE inhibitors are used increasingly 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 the people who continue ACE inhibitors while on dialysis may be able to go off dialysis in 12 to 18 months.
• Several drugs are now available to treat pulmonary hypertension. Previously, pulmonary hypertension was associated with a poor outcome, but medications such as prostacyclins, endothelin-receptor antagonists, and phosphodiesterase inhibitors—epoprostenol sodium, bosentan, and sildenafil—have increased the quality of life and life expectancy for people with this dangerous form of lung damage.

Other studies are examining the following:

• The identification of several early immune system targets in scleroderma. Building on this research, scientists are continuing to look for ways to treat scleroderma earlier, before it has a chance to cause irreparable damage.
• The theory that scleroderma is a more aggressive disease associated with more internal organ damage and a worse prognosis in non-Caucasians. Researchers believe that although factors related to both genetics and socioeconomic status may play a role, autoantibodies may be the primary reason that African Americans have such severe disease. A current study is examining that theory. Researchers hope that by better understanding the factors involved in scleroderma, they can design interventions that would improve the course and outcome of the disease.
• 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 a way to control the problem.
• Studies have shown that certain chemicals called cytokines, made from cells in the body, enhance the development of increased collagen. New agents that counteract these cytokines may be helpful in preventing skin thickening.
• 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. Scientists are also working to establish mouse models for other problems related to scleroderma. These models will make it easier to understand these problems and develop treatments for them.

Scleroderma research continues to advance as scientists and doctors learn more about how the disease develops and its underlying mechanisms with the goal of translating basic science findings into improved treatment and patient care.

References:

• The National Institute on Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, U.S. Department of Human and Health Services.

 

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