Esclerodermia

Research is finding better ways to treat symptoms, prevent organ damage and improve the quality of life for people with scleroderma. Multidisciplinary research has also provided new clues for understanding the disease, which is an important step toward prevention and cure.

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.

Here’s what some recent studies have found:

• 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 study suggested that treating lung problems early with this immunosuppressive drug may help prevent further damage and increase chances of survival. Clinical trials assessing the effectiveness of other medications for lung fibrosis are ongoing.
• ACE inhibitors are used increasingly for scleroderma-related kidney problems. ACE inhibitors have greatly reduced the risk of kidney failure in people with scleroderma.
• Several drugs are now available to treat pulmonary hypertension. Previously, pulmonary hypertension was associated with a poor outcome, but medications have increased the quality of life and life expectancy for people with this dangerous form of lung damage.

Here are some areas of ongoing research:

• Researchers are looking at the complex immune system trying to determine why the body creates antibodies against its own tissues. Identifying where and when the antibodies are created and how they function could lead researchers to find targets for new therapies. Researchers are already trying new therapies, including those that suppress specific parts of the immune system.
• Studies continue to find the genes that predispose a person to getting scleroderma. Those genes may be targets for future therapies. Researchers are also trying to find genes (and other indicators) that could tell doctors who will develop what type of scleroderma. This could someday help doctors identify who might benefit from specific therapies. Additionally, since genes are not the only factor involved in scleroderma, researchers are looking for environmental factors that might trigger the disease in those who are genetically predisposed.
• Some scientists are trying to make better drugs to treat scleroderma and its symptoms. Other researchers are testing existing drugs to see if they are successful in delaying cardiovascular changes.
• Experiments using mice have proved valuable in past research on scleroderma. Further research using existing strains of mice continues.
• Researchers are looking into the many factors that lead to fibrosis to see if they can block its development.
• Studies have shown that certain chemicals called cytokines, made from cells in the body, contribute to inflammation. Researchers are trying to determine how cytokines contribute to the disease process.