A chronic autoimmune disease of the skin, called scleroderma, is not contagious or infectious and often not harmful. However, when this connective tissue disorder involves any internal organs, it can become dangerous. This is why scientists from Duke University developed a new, more aggressive way to treat scleroderma: a stem cell transplant procedure.
"In these severe cases, conventional drug therapies are not very effective long-term, so new approaches are a priority,” explained lead author Keith Sullivan, MD.
Scleroderma is normally characterized by varying levels of skin hardening, affecting around 300,000 Americans. Scientists know that it is caused by an overproduction of collagen, but not much else is known about how it develops. There is no known cure for scleroderma.
In the new Duke study, researchers tested the new stem cell therapy in a group of 36 scleroderma patients, who also received high-dose chemotherapy and whole-body radiation. The stem cell transplant therapy is designed to replace the patient’s defective immune system that is contributing to scleroderma with stem cells “treated to eliminate self-reacting lymphocytes.” Lymphocytes are cells of the immune system, and when they react with host cells, often called “self” cells, various autoimmune diseases can develop.
The outcomes of the patients receiving the transplant were compared to outcomes of 39 scleroderma patients who did not receive the experimental therapy but instead received a drug called cyclophosphamide, used to suppress the immune system as a treatment for scleroderma.
At the end of the study, which lasted for ten years, researchers found that transplant patients were less likely to continue using anti-scleroderma drugs (nine percent) than patients who did not receive stem cells (44 percent). Overall, survival also benefited the transplant patients most of all.
"Patients and their doctors should carefully weigh the pros and cons of intensive treatment with stem cell transplant, but this may hopefully set a new standard in this otherwise devastating autoimmune disease," Sullivan said. "These advances show the value of medical research and clinical trials in finding better therapies to advance health."
The present study was published in the New England Journal of Medicine.