CD19-directed chimeric antigen receptor T cell therapy in Waldenstrom macroglobulinemia: a preclinical model and initial clinical experience.

ABSTRACT:

Background

Waldenström macroglobulinemia (WM) is an incurable disease and, while treatable, can develop resistance to available therapies and be fatal. Chimeric antigen receptor (CAR) T cell therapy directed against the CD19 antigen has demonstrated efficacy in relapsed or refractory B lymphoid malignancies, and is now approved for B cell acute lymphoblastic leukemia and certain B cell lymphomas. However, CAR T therapy has not been evaluated for use in WM.

Methods and results

We performed preclinical studies demonstrating CAR T cell activity against WM cells in vitro, and developed an in vivo murine model of WM which demonstrated prolonged survival with use of CAR T therapy. We then report the first three patients with multiply relapsed and refractory WM treated for their di

SUBMITTER: Palomba ML 

PROVIDER: S-EPMC8852764 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature