Therapeutic Antibodies in Neurological Diseases: Witnessing the Continuation of the Impressive Success in Neuro-Immunotherapies

Dalakas, Marinos C.; Wiendl, Heinz

doi:10.1007/s13311-022-01266-z

Cited by 2 publications

(1 citation statement)

References 20 publications

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“…In most patients, antibodies against amphiphysin or glutamic acid decarboxylase (GAD) can be detected ( 1 ). The antineuronal immunopathology including autoantibodies and cellular mechanisms specifically targeting GABAergic inhibitory pathways and synaptic signaling machinery are believed to contribute to pathogenesis ( 2 , 3 ). SPS associated with antibodies against amphiphysin is also often accompanied by the occurrence of neoplastic disease ( 1 ).…”

Section: Introductionmentioning

confidence: 99%

Successful use of anti-CD19 CAR T cells in severe treatment-refractory stiff-person syndrome

Faissner,

Motte,

Sgodzai

et al. 2024

Proc. Natl. Acad. Sci. U.S.A.

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Treatment with autologous chimeric antigen receptor (CAR) T cells has emerged as a highly effective approach in neuroimmunological disorders such as myasthenia gravis. We report a case of successful anti-CD19 CAR T cell use in treatment-refractory stiff-person syndrome (SPS). To investigate clinical and immunological effects of anti-CD19 CAR T cell use in treatment-refractory SPS, a 69-y-old female with a 9-y history of treatment-refractory SPS with deteriorating episodes of stiffness received an infusion of autologous anti-CD19 CAR T cells (KYV-101) and was monitored clinically and immunologically for more than 6 mo. CAR T cell infusion resulted in reduced leg stiffness, drastic improvement in gait, walking speed increase over 100%, and daily walking distance improvement from less than 50 m to over 6 km within 3 mo. GABAergic medication (benzodiazepines) was reduced by 40%. KYV-101 CAR T cells were well tolerated with only low-grade cytokine release syndrome. This report of successful use of anti-CD19 CAR T cells in treatment-refractory SPS supports continued exploration of this approach in SPS and other B cell–related autoimmune disorders.

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