Stem-Cell Therapy Enables Long-Term Remission for Neuromyelitis Optica Spectrum Disorder Patients

A man and a woman suffering from a rare, life-threatening autoimmune disorder have maintained remission for over 15 years following stem-cell therapy. Published in With, the findings indicate potential for expanded clinical trials to evaluate this experimental approach, according to researchers.

The individuals were diagnosed with neuromyelitis optica spectrum disorder (NMOSD), a condition where immune cells generate antibodies targeting the spinal cord and optic nerve. Symptoms, including acute vision loss, intense eye pain, vomiting, and paralysis, occur in episodic flare-ups. Conventional therapies, which rely on continuous medication to suppress immune activity, failed to control the disease in these patients.

Following allogeneic hematopoietic stem-cell transplants—a procedure typically used for blood cancers and sickle-cell disease—both participants experienced significant neurological improvement. The man regained full functionality, resumed daily activities, and had children, while the woman restored partial arm mobility and discontinued symptom-management medications.

Dr. Jiao Jiao Li, a biomedical engineer at the University of Technology Sydney, noted the treatment’s long-term efficacy: “It hasn’t eradicated NMOSD entirely, but it has effectively managed its progression over this extended period.”

The man received stem cells from his sister in 2009, while the woman underwent an unrelated donor transplant in 2010. Both underwent a single infusion of donor hematopoietic stem cells after preparatory chemotherapy with fludarabine and treosulfan and targeted B-cell depletion with monoclonal antibodies.

To prevent graft-versus-host disease, recipients were administered antibodies and immunosuppressants. Neither developed NMOSD-associated antibodies post-transplant, and both achieved full immunomodulation, per the study. Dr. Bruce Milthorpe of the same institution highlighted the significance: “Sustaining symptom-free status for years is a major breakthrough.”

The therapy uniquely replaces the entire immune system, unlike autologous versions that recycle existing cells. For NMOSD, eliminating antibody-producing B cells is critical, as residual cells could perpetuate autoimmunity—a challenge addressed by allogeneic regimens, according to Dr. Li.

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