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Mar Guasp is a recipient of Juan Rodes grant (JR23/00030) from the Instituto de Salud Carlos III, co-financed by Fondo Social Europeo Plus.

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CD19-Directed CAR T-Cells in a Patient With Refractory MOGAD

Publicado en:Neurology-Neuroimmunology & Neuroinflammation. 11 (5): e200292- - 2024-09-01 11(5), DOI: 10.1212/NXI.0000000000200292

Autores: Cabrera-Maqueda, Jose Maria; Sepulveda, Maria; Garcia, Raquel Ruiz; Munoz-Sanchez, Guillermo; Martinez-Cibrian, Nuria; Ortiz-Maldonado, Valentin; Lorca-Arce, Daniel; Guasp, Mar; Llufriu, Sara; Martinez-Hernandez, Eugenia; Armangue, Thais; Fonseca, Elianet G; Alba-Isasi, Maria Teresa; Delgado, Julio; Dalmau, Josep; Juan, Manel; Saiz, Albert; Blanco, Yolanda

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ObjectivesIn MOG antibody-associated disease (MOGAD), relapse prevention and the treatment approach to refractory symptoms are unknown. We report a patient with refractory MOGAD treated with CD19-directed CAR T-cells.MethodsCD19-directed CAR T-cells (ARI-0001) were produced in-house by lentiviral transduction of autologous fresh leukapheresis and infused after a conventional lymphodepleting regimen.ResultsA 18-year-old man developed 2 episodes of myelitis associated with serum MOG-IgG, which were followed by 6 episodes of left optic neuritis (ON) and sustained the presence of MOG-IgG over 6 years despite multiple immunotherapies. After the sixth episode of ON, accompanied by severe residual visual deficits, CAR T-cell treatment was provided without complications. Follow-up of cell counts showed complete depletion of CD19+ B cells at day +7; reconstituted B cells at day +141 showing a na & iuml;ve B-cell phenotype, and low or absent memory B cells and plasmablasts for 1 year. MOG-IgG titers have remained undetectable since CAR T-cell infusion. The patient had an early episode of left ON at day +29, when MOG-IgG was already negative, and since then he has remained free of relapses without immunotherapy for 1 year.DiscussionThis clinical case shows that CD19-directed CAR T-cell therapy is well-tolerated and is a potential treatment for patients with refractory MOGAD.Classification of EvidenceThis provides Class IV evidence. It is a single observational study without controls.

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