Donor stem-cell transplant keeps two neuromyelitis optica patients in remission over 15 years
Two people with neuromyelitis optica, a disease where immune antibodies attack the spinal cord and optic nerve, have stayed in remission more than 15 years after a single donor stem-cell transplant. Standard drugs had failed both. It is the first use of the approach for this cond
Two people with neuromyelitis optica spectrum disorder, a rare and potentially fatal autoimmune disease in which the immune system produces antibodies that attack the spinal cord and the nerve connecting the eye to the brain, have remained in remission for more than 15 years after receiving a donor stem-cell transplant. The results were reported in the journal Med by a team including Massimo Filippi, a neurologist at IRCCS San Raffaele Hospital in Milan. The disease tends to strike in episodes lasting days or months, causing eye pain, vision loss, vomiting, and weakness or paralysis in the arms and legs. Ongoing medication can hold these episodes back in many patients, but it had stopped working for both of these individuals. The treatment, called allogeneic haematopoietic stem-cell transplant, replaces a person's immune system with blood stem cells from a donor. The same procedure already treats some cancers, sickle-cell disease and other blood disorders. The man received cells from his sister in 2009 and the woman received cells from an unrelated donor the following year, each in a single infusion. Before the transplant, both underwent chemotherapy with fludarabine and treosulfan plus a monoclonal antibody to strip out the B cells driving the disease. After treatment, the man's neurological function improved enough that he resumed a normal life and went on to have two children. The woman regained use of her arms and no longer needs medication to control symptoms. Neither developed the antibodies linked to the disease again. Researchers stop short of calling it a cure and note this is the first reported use of the approach for this specific condition, in just two patients. They say the long remission is encouraging and argue it justifies a larger clinical trial. Source: Nature, reporting on a study in Med.