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.
Overall, 85% of patients (95% CI, 68.1-94.9) responded to BCMA
CAR-T therapy, with 45% (15 patients) achieving complete responses. Among the
15 patients with complete responses, 6 have relapsed. Complete responses
occurred across all infusion doses, and responses were associated with CAR-T
cell expansion. Negative minimal residual disease was detected for all 16
patients for whom response was achieved and minimal residual disease was
evaluable. Patients had a median progression-free survival of 11.8 months (95%
A phase 2 trial (KarMMa-2) is under way (ClinicalTrials.gov
- Raje N, Berdeja J, Lin Y, et al. Anti-BCMA CAR T-cell
therapy bb2121 in relapsed or refractory multiple myeloma. N Engl J Med. 2019;380(18):1726-1737.
publishes bb2121 Phase 1 data in patients with multiple myeloma [news
release]. Summit, NJ, and Cambridge, MA: Celgene Corporation and bluebird bio,
Inc.; May 2, 2019. Accessed May 8, 2019.
Originally published: https://www.cancertherapyadvisor.com/home/cancer-topics/multiple-myeloma/multiple-myeloma-bcma-cart-bb2121-appears-safe-effective-treatment/