医学
耐火材料(行星科学)
嵌合抗原受体
细胞因子释放综合征
内科学
多发性骨髓瘤
淀粉样变性
淀粉样变性
推车
肿瘤科
临床试验
胃肠病学
外科
癌症
免疫疗法
免疫学
抗体
免疫球蛋白轻链
天体生物学
机械工程
工程类
物理
作者
Shlomit Kfir‐Erenfeld,Nathalie Asherie,Sigal Grisariu,Batia Avni,Eran Zimran,Miri Assayag,Tatyana Dubnikov Sharon,Marjorie Pick,Eyal Lebel,Adir Shaulov,Yaël Cohen,Irit Avivi,Cyrille J. Cohen,Polina Stepensky,Moshe E. Gatt
标识
DOI:10.1158/1078-0432.ccr-22-0637
摘要
Abstract Purpose: AL amyloidosis (AL) treatments are generally based on those employed for multiple myeloma. Anti–B-cell maturation antigen (BCMA) chimeric antigen receptor T (CART)-cell therapy, already approved for multiple myeloma, might be too toxic for patients with AL. Experimental Design: Here we describe the ex vivo applicability of a novel in-house, academic anti-BCMA CAR construct on AL primary cells, as well as the safety and efficacy in 4 patients with relapsed/refractory (RR) primary AL, treated in a phase I clinical trial (NCT04720313). Results: Three had MAYO stage IIIa cardiac involvement at enrollment. The treatment proved relatively safe, with a short and manageable grade 3 cytokine release syndrome evident in 2 patients and no neurotoxicity in any. Cardiac decompensations, observed in 2 patients, were also short and manageable. The overall hematologic response and complete response rates were observed in all patients with an organ response evident in all four. Within a median follow-up period of 5.2 (2.5–9.5) months, all 4 patients maintained their responses. Conclusions: BCMA-CART cells provide a first proof-of-concept that this therapy is safe enough and highly efficacious for the treatment of patients with advanced, RR AL.
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