New England Journal of Medicine · 2019 · 1.7K citations · 26 references
MedicineImmunologyOncologyCancer ImmunologyCell TherapyImmunotherapyB Cell BiologyB-cell ResponsesCancer ImmunotherapyChimeric Antigen Receptor T-cell TherapyHematologic Malignancy TherapyMultiple MyelomaB Cell-targeted ImmunotherapyChimeric Antigen Receptor DesignMultiple Myeloma Cellular TherapyMultiple Myeloma TherapyMultiple Myeloma TreatmentRefractory Multiple MyelomaRelapsed Multiple MyelomaT-cell Therapy
BACKGROUND: Preclinical studies suggest that bb2121, a chimeric antigen receptor (CAR) T-cell therapy that targets B-cell maturation antigen (BCMA), has potential for the treatment of multiple myeloma. METHODS: CAR+ T cells in the expansion phase. Patients had received at least three previous lines of therapy, including a proteasome inhibitor and an immunomodulatory agent, or were refractory to both drug classes. The primary end point was safety. RESULTS: nucleated cells). CAR T-cell expansion was associated with responses, and CAR T cells persisted up to 1 year after the infusion. CONCLUSIONS: We report the initial toxicity profile of a BCMA-directed cellular immunotherapy for patients with relapsed or refractory multiple myeloma. Antitumor activity was documented. (Funded by Bluebird Bio and Celgene; CRB-401 ClinicalTrials.gov number, NCT02658929.).
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