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

• 成果类型: Article
• 署名作者: Faissner, Simon; Motte, Jeremias; Sgodzai, Melissa; Geis, Christian; Haghikia, Aiden; Mougiakakos, Dimitrios; Borie, Dominic; Schroers, Roland; Gold, Ralf
• 署名单位: Ruhr University Bochum; Friedrich Schiller University of Jena; Otto von Guericke University; Otto von Guericke University; Ruhr University Bochum
• 刊物名称: PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
• ISSN/ISSBN: 0027-12582
• DOI: 10.1073/pnas.2403227121
• 发表日期: 2024-06-25
• 关键词: therapy; disease

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