A new clinical trial evaluating the safety and efficacy of chimeric antigen receptor (CAR)-T cell therapy in patients with relapsed or refractory neuromyelitis optica spectrum disorder (NMOSD) is now recruiting participants.

The open-label, single-arm, single-center, interventional, phase 1 study will also evaluate the pharmacokinetics of the treatment.

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An estimated 9 patients with recurrent or refractory NMOSD, who were 18 to 60 years of age, will be given CAR-T cell injections.

The primary outcome measures will be dose-limited toxicity, adverse events and serious adverse events, and the maximum tolerable dose of the treatment. 

Secondary outcome measures are changes in serum aquaporin 4 antibodies (AQP4-IgG) titer after infusion, the annual recurrence rate of NMOSD, changes in the expanded disability status scale score from baseline, active lesions seen on magnetic resonance imaging (MRI), and changes in optimal corrected vision, nerve fiber layer around the retinal papilla, plexiform layer of macular ganglion cells, and flash visual evoked potential. 

The trial is estimated to start on April 30, 2023, and be completed on April 30, 2026. It is currently recruiting participants in The First Affiliated Hospital of Medical College of Zhejiang University in China.

NMOSD is a rare autoimmune disease of the central nervous system characterized by chronic inflammation especially affecting the optic nerve and leading to repeated episodes of severe optic neuritis and/or myelitis. Most patients with the disease have autoantibodies against the AQP4 water channel found on astrocytes.

The treatment of NMOSD focuses on achieving disease remission, reducing symptoms associated with acute flares and relapses, preventing relapses, and managing disability. 

CAR T cell therapy is a type of immunotherapy that uses engineered T cells that can locate and destroy specific cells more effectively. It has already been used to treat hematologic malignancies and autoimmune diseases and is thought to also be efficient in patients with NMOSD.


Evaluate the safety and efficacy of CAR-T cells in the treatment of R/R neuromyelitis optica. US National Library of Medicine. Updated April 25, 2023. Accessed April 26, 2023.