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

被引:22
作者
Faissner, Simon [1 ]
Motte, Jeremias [1 ]
Sgodzai, Melissa [1 ]
Geis, Christian [2 ]
Haghikia, Aiden [3 ]
Mougiakakos, Dimitrios [4 ]
Borie, Dominic [5 ]
Schroers, Roland [6 ]
Gold, Ralf [1 ]
机构
[1] Ruhr Univ Bochum, St Josef Hosp, Dept Neurol, D-44791 Bochum, Germany
[2] Jena Univ Hosp, Dept Neurol, D-07747 Jena, Germany
[3] Otto von Guericke Univ, Med Fac, Dept Neurol, D-39120 Magdeburg, Germany
[4] Otto von Guericke Univ, Med Fac, Dept Hematol, D-39120 Magdeburg, Germany
[5] Kyverna Therapeut, Emeryville, CA 94608 USA
[6] Ruhr Univ Bochum, Knappschaftskrankenhaus Bochum, Dept Haematol & Oncol, D-44892 Bochum, Germany
关键词
CART cells; chimeric antigen receptor; person syndrome; neuroimmunological disorder; B cell autoimmunity; THERAPY; DISEASE;
D O I
10.1073/pnas.2403227121
中图分类号
O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];
学科分类号
07 ; 0710 ; 09 ;
摘要
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, ing 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. walking speed increase over 100%, and daily walk
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页数:3
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