Can stem cell transplants help?
In a bold attempt to control the disabling and often fatal autoimmune disease scleroderma, physicians at Duke are leading a national study to test whether stem cell transplants can reconstruct defective immune systems. If successful, the therapy would represent the first therapy ever to treat and potentially reverse the disease itself, not just alleviate its symptoms. In people with scleroderma, the immune system attacks the body’s connective tissues that support the skin and internal organs. These patients experience varying degrees of pain, inflammation, hardened skin, and organ failure. The standard therapy is the chemotherapy drug cyclophosphamide, which suppresses the immune system. The drug alleviates symptoms but doesn’t alter the course of the disease — about half of patients with severe organ involvement die within five years of diagnosis, despite treatment. “We are hoping that stem cell transplantation will actually lessen or eradicate the defective immune response that init
