Fifteen years of remission after one treatment
A man and a woman with neuromyelitis optica spectrum disorder (NMOSD)—a rare autoimmune disease where the immune system attacks the spinal cord and optic nerve—have been in remission for more than 15 years after receiving a single stem-cell transplant, researchers reported in Nature. The disease can cause blindness, paralysis, and is potentially fatal.
How donor stem cells reset the immune system
The allogeneic hematopoietic stem-cell transplant used donor stem cells collected from another person's blood. Before the transplant, patients received chemotherapy drugs fludarabine and treosulfan along with a monoclonal antibody to eliminate the B cells producing the harmful antibodies. Unlike autologous transplants that use a patient's own stem cells, this donor-cell approach provided a completely new immune system, eliminating the underlying autoimmune mechanism.
Implications for autoimmune treatment
Co-author Massimo Filippi, a neurologist at IRCCS San Raffaele Hospital, called this the first use of this therapy for NMOSD. While allogeneic transplants carry risks including graft-versus-host disease, the 15-year durability of remission suggests the approach could transform treatment paradigms for severe autoimmune conditions, potentially offering a cure rather than lifelong disease management.