Treatment of concomitant myasthenia gravis and Lambert-Eaton myasthenic syndrome with autologous CD19-targeted CAR T cells

被引:13
|
作者
Motte, Jeremias [1 ]
Sgodzai, Melissa [1 ]
Schneider-Gold, Christiane [1 ]
Steckel, Nina [2 ]
Mika, Thomas [2 ]
Hegelmaier, Tobias
Borie, Dominic [4 ]
Haghikia, Aiden [3 ]
Mougiakakos, Dimitrios [5 ]
Schroers, Roland [2 ]
Gold, Ralf [1 ]
机构
[1] Ruhr Univ Bochum, St Josef Hosp Bochum, Dept Neurol, D-44791 Bochum, Germany
[2] Ruhr Univ Bochum, Dept Hematol & Oncol, Knappschaftskrankenhaus, D-44892 Bochum, Germany
[3] Otto Von Guericke Univ, Dept Neurol, D-39120 Magdeburg, Germany
[4] Kyverna Therapeut, Emeryville, CA 94608 USA
[5] Otto von Guericke Univ, Dept Hematol & Oncol, D-39120 Magdeburg, Germany
关键词
THERAPY;
D O I
10.1016/j.neuron.2024.04.014
中图分类号
Q189 [神经科学];
学科分类号
071006 ;
摘要
Myasthenia gravis (MG) and Lambert -Eaton myasthenic syndrome (LEMS) are autoimmune disorders affecting neuromuscular transmission. Their combined occurrence is rare, and treatment remains challenging. Two women diagnosed with concomitant MG/LEMS experienced severe, increasing disease activity despite multiple immunotherapies. Anti-CD19 chimeric antigen receptor (CAR) T cells have shown promise for treating autoimmune diseases. This report details the safe application of anti-CD19 CAR T cells for treating concomitant MG/LEMS. After CAR T cell therapy, both patients experienced rapid clinical recovery and regained full mobility. Deep B cell depletion and normalization of acetylcholine receptor and voltage -gated calcium channel N -type autoantibody levels paralleled major neurological responses. Within 2 months, both patients returned to everyday life, from wheelchair dependency to bicycling and mountain hiking, and remain stable at 6 and 4 months post -CAR T cell infusion, respectively. This report highlights the potential for antiCD19 CAR T cells to achieve profound clinical effects in the treatment of neuroimmunological diseases.
引用
收藏
页码:1757 / 1763.e2
页数:10
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