Donor stem-cell transplants hold a severe autoimmune disease in remission for 15 years
Two patients with neuromyelitis optica spectrum disorder (NMOSD) — a severe autoimmune condition in which immune cells attack the spinal cord and optic nerve, causing vision loss, pain and paralysis — have stayed in remission for more than 15 years after receiving donor stem-cell transplants, according to a case report published in the journal Med by Orofino and colleagues and described by Nature .
Both patients had failed standard medication. They received allogeneic haematopoietic stem-cell transplants — donor cells drawn from blood, one from the patient’s sister and one from an unrelated donor — after chemotherapy and immunosuppression to prevent rejection. The man, treated in 2009, regained neurological function, returned to normal life and later fathered two children; the woman, treated in 2010, recovered arm function and stopped her symptom-management drugs.
The cases are notable because the transplants used donor cells rather than the patient’s own. As one researcher put it, the approach “completely replaces the person’s immune system,” which may work better for autoimmune disease than autologous transplants that reinfuse a patient’s own cells. The caveat is scale: this is a two-patient report describing outcomes from procedures performed more than a decade ago, not a controlled trial. Allogeneic transplants also carry serious risks, including graft-versus-host disease, which keeps the procedure reserved for the most refractory cases pending larger studies.