The following summary is “Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis ” published in the December 2022 issue of Oncology by Kfir-Erenfeld et al.

Treatments for AL amyloidosis (AL) are typically derived from those used for multiple myeloma. Chimeric antigen receptor T (CART)-cell therapy against anti–B-cell maturation antigen (BCMA), which has already been licensed for treating multiple myeloma, may be too hazardous for patients with AL. In this paper, researchers present the ex vivo applicability of a unique academic anti-BCMA CAR design on AL primary cells, as well as the safety and efficacy of the treatment in 4 patients with relapsed or refractory (RR) primary AL who were treated in phase I clinical trial (NCT04720313).

At the time of enrolment, 3 patients had MAYO stage IIIa cardiac involvement. The treatment was found to be reasonably safe, with only 2 patients experiencing a mild grade 3 cytokine release syndrome that lasted only a short amount of time and was easily managed and with no patients showing any signs of neurotoxicity. 2 patients experienced cardiac decompensations, both of which lasted only a short time and were easily managed. 

All 4 patients showed evidence of an organ response, consistent with the overall hematologic response and full response rates found in all patients. All 4 patients could keep their responses throughout the follow-up period, which lasted a median of 5.2 (2.5–9.5) months. BCMA-CART cells offer the first proof-of-concept that this therapy can treat patients with advanced RR AL in a msafe and effective manner.