Stem-Cell Transplant Delivers 15-Year Remission in 2 Severe NMOSD Patients

3 articles · Updated · Scientific American · Jun 22

More than 15 years after a single donor stem-cell infusion, 2 people with severe neuromyelitis optica spectrum disorder remain in remission after standard drugs had failed.
The allogeneic transplants—performed in 2009 and 2010 after chemotherapy and immune-cell depletion—appeared to replace the patients’ disease-driving immune systems; neither later developed NMOSD-linked antibodies.
Function also improved: the man resumed a normal life and had 2 children, while the woman regained better arm use and no longer needs symptom-control medication.
Risks remain substantial, including graft-versus-host disease, infections, antibody deficiency and one case of bladder cancer, leaving researchers to say the approach should be limited to selected younger patients.
Because the report covers only 2 cases, scientists say the results are not proof of a cure but strong enough to justify a larger clinical trial.

Sources

Scientific American15h ago

Stem-Cell Transplant Induces 15-Year Remission in Severe Autoimmune NMOSD Patients

Medical Xpress15h ago

Two patients with severe autoimmune disease remain relapse-free for over 15 years after stem cell transplant

Scientific American15h ago

Stem cells banish severe autoimmune disease for 15 years | Scientific American

4 Sources

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15-Year Remission in Severe NMOSD: Allogeneic Stem Cell Transplantation Offers New Hope and Challenges

Overview

A major milestone has been reached in the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD), as two patients with severe, treatment-resistant forms of the disease have achieved 15 years of remission after undergoing allogeneic hematopoietic stem cell transplantation (HSCT). This long-term remission offers new hope for those battling this debilitating autoimmune disorder and serves as important proof-of-concept that allogeneic HSCT can fundamentally change the disease course in severe cases. However, since the study involved only two patients, more research is needed to determine if this treatment will be effective for all NMOSD patients.

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