Scleroderma, part of an insidious family of diseases where the immune system attacks a patient’s own body, sometimes enough to kill. So far, there is no treatment of the disease. Now the researchers in U.S. and in Europe are looking at methods to re-set immunity for patients with severe scleroderma.
Bari Martz is one such patient who suffered from the disease. She started gasping for breath, and her joints stiffened so that she couldn’t even open her hands.
Desperate of getting rid of the disease, she took a gamble. Doctors stored stem cells from her blood and then wiped out her faulty immune system. Her reinfused stem cells seem to have let a healthy new immune system take root, stopping more damage and, nearly two years later, letting her lungs and joints heal enough for better function.
About 300,000 Americans have various forms of scleroderma, often confined to the skin.But a third have systemic scleroderma, the most severe form that invades internal organs. Only the cancer drug cyclophosphamide is proven to slow severe scleroderma, but its effects are modest. About half of severely affected patients die in five years.
Enter stem cell transplants. Similar to a bone marrow transplant, it’s a risky treatment usually reserved for leukemia. A type of stem cell that generates immune-system cells is culled from patients’ blood, and then radiation or chemotherapy or both destroy circulating immune cells — leaving the person vulnerable to life-threatening infections until the stem cells are returned and produce again.