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

Research output: Contribution to journalArticlepeer-review

13 Scopus citations


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 languageEnglish
Pages (from-to)5156-5166
Number of pages11
JournalClinical Cancer Research
Issue number23
StatePublished - 1 Dec 2022
Externally publishedYes


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