BCMA CAR-T Cell Therapy Achieves 100% ORR in Latest Clinical Research Findings
- Elva Chen
- Oct 21
- 2 min read
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Recently, the international hematology journal Blood Advances published a prospective clinical study by Lu Daopei Medical Team, titled "Efficacy and safety of BCMA nanobody CAR T-cell therapy in relapsed or refractory plasma cell myeloma," reporting breakthrough clinical results of BCMA-targeted nanobody-based CAR-T cell therapy in heavily pretreated refractory/relapsed plasma cell neoplasms.
Research Background
Patients with relapsed/refractory multiple myeloma (R/R MM), particularly those with extramedullary disease, central nervous system (CNS) involvement, or high-risk genetic abnormalities, have a very poor prognosis. B-cell maturation antigen (BCMA) CAR-T cell therapy offers new hope, but efficacy of traditional single-chain variable fragment (scFv) constructed CAR-T is limited by antigen loss, immunogenicity, and cell persistence. This study evaluated the safety and efficacy of a novel dual nanobody (dual-VHH) BCMA CAR-T cell therapy.
Research Methods
The study enrolled 27 patients with complex/high-risk R/R plasma cell neoplasms, including 22 with multiple myeloma, 4 with plasma cell leukemia, and 1 with anaplastic plasmacytoma. Among them, 11 had multiple extramedullary lesions (including 3 with CNS involvement) and 11 had high-risk genetic abnormalities (e.g., TP53 mutations). Patients received an intravenous infusion of BCMA CAR-T cells at a dose of 1×10⁶/kg (range: 0.3–2×10⁶/kg). Primary endpoints were overall response rate (ORR) and safety; secondary endpoints included depth of response, duration of response (DOR), progression-free survival (PFS), and overall survival (OS).
Research Results
Results showed that three months post-BCMA CAR-T cell infusion, the Overall Response Rate (ORR) was 100%, with a deep response rate (CR + VGPR) of 81.5%. The median duration of response (DOR) was 11 months (range: 2–36 months). In high-risk genetic abnormality patients, the 3-month CR rate was 72.7%, and the 1-year PFS was 65.5%. All three patients with CNS involvement responded, with two achieving CR. The patient with anaplastic plasmacytoma achieved bone marrow MRD negativity, significant tumor reduction, and recovery of motor function after treatment.
Regarding safety, the primary adverse reactions were low-grade cytokine release syndrome (CRS) and mild neurotoxicity. The main complication was infection, which could be managed with active intervention.
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