{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Palomba ML"],"funding":["Carson Family Charitable Trust","Emerald Foundation","Canada Club of New York, Annual Terry Fox Run for Cancer Research","Memorial Sloan-Kettering Cancer Center","William Lawrence and Blanche Hughes Foundation","NCI NIH HHS","National Institutes of Health","Memorial Sloan-Kettering Cancer Center Experimental Therapeutics Center","NIH HHS"],"pagination":["e004128"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8852764"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["10(2)"],"pubmed_abstract":["<h4>Background</h4>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.<h4>Methods and results</h4>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"],"journal":["Journal for immunotherapy of cancer"],"pubmed_title":["CD19-directed chimeric antigen receptor T cell therapy in Waldenstrom macroglobulinemia: a preclinical model and initial clinical experience."],"pmcid":["PMC8852764"],"funding_grant_id":["K08CA241400-01","U54 OD020355","P30 CA008748","K08 CA241400","U54 OD020355-01","R01 CA138738-05","R01 CA138738"],"pubmed_authors":["Dogan A","Riviere I","Palomba ML","Brentjens RJ","Sethi S","Park JH","Senechal B","Monette S","Sadelain M","Smith EL","Roshal M","Qualls D","Wang X"],"additional_accession":[]},"is_claimable":false,"name":"CD19-directed chimeric antigen receptor T cell therapy in Waldenstrom macroglobulinemia: a preclinical model and initial clinical experience.","description":"<h4>Background</h4>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.<h4>Methods and results</h4>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","dates":{"release":"2022-01-01T00:00:00Z","publication":"2022 Feb","modification":"2026-06-07T03:18:56.717Z","creation":"2025-04-06T13:56:13.17Z"},"accession":"S-EPMC8852764","cross_references":{"pubmed":["35173030"],"doi":["10.1136/jitc-2021-004128"]}}