Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis

Shlomit Kfir-Erenfeld; Nathalie Asherie; Sigal Grisariu; Batia Avni; Eran Zimran; Miri Assayag; Tatyana Dubnikov Sharon; Marjorie Pick; Eyal Lebel; Adir Shaulov; Yael C. Cohen; Irit Avivi; Cyrille J. Cohen; Polina Stepensky; Moshe E. Gatt

doi:10.1158/1078-0432.CCR-22-0637

Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis

Shlomit Kfir-Erenfeld, Nathalie Asherie, Sigal Grisariu, Batia Avni, Eran Zimran, Miri Assayag, Tatyana Dubnikov Sharon, Marjorie Pick, Eyal Lebel, Adir Shaulov, Yael C. Cohen, Irit Avivi, Cyrille J. Cohen, Polina Stepensky, Moshe E. Gatt

The Mina and Everard Goodman Faculty of Life Sciences

Research output: Contribution to journal › Article › peer-review

3 Scopus citations

Abstract

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.

Original language	English
Pages (from-to)	5156-5166
Number of pages	11
Journal	Clinical Cancer Research
Volume	28
Issue number	23
DOIs	https://doi.org/10.1158/1078-0432.CCR-22-0637
State	Published - 1 Dec 2022

Bibliographical note

Funding Information:
This work is supported by generous support from Steinfeld and Cuniff family, the estate of Allan Habelson, and by the Amyloidosis Patient Association of Israel. C.J. Cohen is supported by the Adelis Foundation and the Israel Science Foundation (646/20).

Publisher Copyright:
©2022 American Association for Cancer Research.

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10.1158/1078-0432.CCR-22-0637

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Kfir-Erenfeld, S., Asherie, N., Grisariu, S., Avni, B., Zimran, E., Assayag, M., Sharon, T. D., Pick, M., Lebel, E., Shaulov, A., Cohen, Y. C., Avivi, I., Cohen, C. J., Stepensky, P., & Gatt, M. E. (2022). Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis. Clinical Cancer Research, 28(23), 5156-5166. https://doi.org/10.1158/1078-0432.CCR-22-0637

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title = "Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis",

abstract = "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.",

author = "Shlomit Kfir-Erenfeld and Nathalie Asherie and Sigal Grisariu and Batia Avni and Eran Zimran and Miri Assayag and Sharon, {Tatyana Dubnikov} and Marjorie Pick and Eyal Lebel and Adir Shaulov and Cohen, {Yael C.} and Irit Avivi and Cohen, {Cyrille J.} and Polina Stepensky and Gatt, {Moshe E.}",

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doi = "10.1158/1078-0432.CCR-22-0637",

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Kfir-Erenfeld, S, Asherie, N, Grisariu, S, Avni, B, Zimran, E, Assayag, M, Sharon, TD, Pick, M, Lebel, E, Shaulov, A, Cohen, YC, Avivi, I, Cohen, CJ, Stepensky, P & Gatt, ME 2022, 'Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis', Clinical Cancer Research, vol. 28, no. 23, pp. 5156-5166. https://doi.org/10.1158/1078-0432.CCR-22-0637

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.

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.

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Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis

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