| Title |
Successful fresh formulation CD19 CAR-T cell therapy for GAD65 antibody-mediated cerebellar ataxia. A case report |
| Authors |
Vaišvilas, Mantas ; Černauskienė, Skirmantė ; Petrosian, David ; Giedraitienė, Nataša ; Stoškus, Mindaugas ; Griškevičius, Laimonas |
| DOI |
10.3389/fimmu.2026.1755797.s003 |
| Full Text |
|
| Is Part of |
Frontiers in immunology.. Lausanne : Frontiers Media SA. 2026, vol. 17, art. no. 1755797, p. [1-5].. eISSN 1664-3224 |
| Keywords [eng] |
autoimmune cerebellar ataxia ; autoimmune encephalitis ; CAR-T cell therapy ; GAD65 antibody ; gene therapy ; immunotherapy |
| Abstract [eng] |
Background: Chimeric antigen receptor T (CAR-T) cell therapy is an effective treatment for treatment-refractory hematological disorders with an acceptable safety profile. In contrast, preliminary reports suggest good efficacy for treatment-refractory autoimmune disorders, including autoimmune nervous system disease, but their safety profile is largely unknown.Objective: To describe the first case of glutamic acid decarboxylase-65 (GAD65) antibody-mediated cerebellar ataxia (CA) successfully treated with CD19 CAR-T cells.Results: A 33-year-old male was diagnosed with GAD65 antibody mediated CA in 2023. Despite treatment with Rituximab and Cyclophosphamide, the patient’s condition worsened with new-onset recurrent falls and increasing vertigo. Ambulation was maintained. CD19 CAR-T cells at a dose of 1 × 106 cells per kilogram of body weight were infused after administration of standard lymphodepleting chemotherapy, resulting in a good serological response with reduction of GAD65 serum titers by 95% at day +90, significant clinical improvement in ataxia at day +30 and no evidence of disease progression at day +270 clinically, radiologically and laboratory-wise. The toxicity was limited to cytokine release syndrome grade 1.Discussion: The favorable clinical response observed in our patient, along with other reports demonstrating preliminary efficacy and limited toxicity, supports further study of CD19 CAR-T cell therapy in GAD65 neurological disorders. |
| Published |
Lausanne : Frontiers Media SA |
| Type |
Journal article |
| Language |
English |
| Publication date |
2026 |
| CC license |
|
