An investigational BCMA-directed chimeric antigen receptor (CAR) T-cell (CAR-T) therapy, bb2121, appeared safe and effective for previously treated patients with relapsed or refractory multiple myeloma in a phase 1 trial (ClinicalTrials.gov identifier: NCT02658929). The trial results were published May 2, 2019, in the New England Journal of Medicine.1
The phase 1 trial, ran by Celgene in collaboration with bluebird bio, Inc., included 33 patients with relapsed or refractory multiple myeloma who had received at least 3 prior lines of therapy. Patients received lymphodepletion with fludarabine and cyclophosphamide followed by a single infusion of bb2121, a BCMA-targeted CAR-T therapy. During the dose-escalation phase, patients received 50×106, 150×106, 450×106, or 800×106 CAR-positive (CAR+) T cells; the expansion phase used 150×106 to 450×106 CAR+ T cells.
Adverse events occurred in all patients, with 97% (32 of 33 patients) having a grade 3 event or higher. Approximately three-quarters of patients (76%) had cytokine release syndrome: 70% had grade 1 or grade 2, and 6% had grade 3. Neurologic toxicity was observed in 42% of patients: 39% had grade 1 or grade 2, and 3% (1 patient) had a reversible grade 4 event.