TY - JOUR
T1 - Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis
AU - Kfir-Erenfeld, Shlomit
AU - Asherie, Nathalie
AU - Grisariu, Sigal
AU - Avni, Batia
AU - Zimran, Eran
AU - Assayag, Miri
AU - Sharon, Tatyana Dubnikov
AU - Pick, Marjorie
AU - Lebel, Eyal
AU - Shaulov, Adir
AU - Cohen, Yael C.
AU - Avivi, Irit
AU - Cohen, Cyrille J.
AU - Stepensky, Polina
AU - Gatt, Moshe E.
N1 - Publisher Copyright:
©2022 American Association for Cancer Research.
PY - 2022/12/1
Y1 - 2022/12/1
N2 - Purpose: AL amyloidosis (AL) treatments are generally based on manageable grade 3 cytokine release syndrome evident in 2 patients those employed for multiple myeloma. Anti–B-cell maturation antigen and no neurotoxicity in any. Cardiac decompensations, observed in (BCMA) chimeric antigen receptor T (CART)-cell therapy, already ap-2 patients, were also short and manageable. The overall hematologic proved for multiple myeloma, might be too toxic for patients with AL. response and complete response rates were observed in all patients Experimental Design: Here we describe the ex vivo applicability of a with an organ response evident in all four. Within a median follow-novel in-house, academic anti-BCMA CAR construct on AL primary up period of 5.2 (2.5–9.5) months, all 4 patients maintained their cells, as well as the safety and efficacy in 4 patients with relapsed/refracresponses. tory (RR) primary AL, treated in a phase I clinical trial (NCT04720313). Conclusions: BCMA-CART cells provide a first proof-of-concept Results: Three had MAYO stage IIIa cardiac involvement at that this therapy is safe enough and highly efficacious for the enrollment. The treatment proved relatively safe, with a short and treatment of patients with advanced, RR AL.
AB - Purpose: AL amyloidosis (AL) treatments are generally based on manageable grade 3 cytokine release syndrome evident in 2 patients those employed for multiple myeloma. Anti–B-cell maturation antigen and no neurotoxicity in any. Cardiac decompensations, observed in (BCMA) chimeric antigen receptor T (CART)-cell therapy, already ap-2 patients, were also short and manageable. The overall hematologic proved for multiple myeloma, might be too toxic for patients with AL. response and complete response rates were observed in all patients Experimental Design: Here we describe the ex vivo applicability of a with an organ response evident in all four. Within a median follow-novel in-house, academic anti-BCMA CAR construct on AL primary up period of 5.2 (2.5–9.5) months, all 4 patients maintained their cells, as well as the safety and efficacy in 4 patients with relapsed/refracresponses. tory (RR) primary AL, treated in a phase I clinical trial (NCT04720313). Conclusions: BCMA-CART cells provide a first proof-of-concept Results: Three had MAYO stage IIIa cardiac involvement at that this therapy is safe enough and highly efficacious for the enrollment. The treatment proved relatively safe, with a short and treatment of patients with advanced, RR AL.
UR - http://www.scopus.com/inward/record.url?scp=85143180343&partnerID=8YFLogxK
U2 - 10.1158/1078-0432.CCR-22-0637
DO - 10.1158/1078-0432.CCR-22-0637
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C2 - 36107221
AN - SCOPUS:85143180343
SN - 1078-0432
VL - 28
SP - 5156
EP - 5166
JO - Clinical Cancer Research
JF - Clinical Cancer Research
IS - 23
ER -
