Department of Hematology, Tianjin Medical University General Hospital, Tianjin, China.
State Key Laboratory of Medicinal Chemical Biology and College of Life Sciences, Nankai University, Tianjin, China.
Br J Haematol. 2023 May;201(3):417-421. doi: 10.1111/bjh.18230. Epub 2022 May 20.
The delivery of bispecific antibodies (BsAbs) targeting B-cell maturation antigen (BCMA) and CD3 using the gene therapy approach is a promising alternative for BsAb administration in patients with multiple myeloma (MM). In the present study, we evaluated the efficacy of this approach using a xenograft model. Tumour growth was significantly delayed in mice treated with single electroporation-enhanced intramuscular injection of plasmid DNA encoding BCMA/CD3 BsAb in contrast to the vehicle control-treated group. Limited toxicity was observed following treatment. This study demonstrates that the gene therapy-based approach for the delivery of BCMA/CD3 BsAb is effective and safe for the treatment of MM.
采用基因治疗方法递送针对 B 细胞成熟抗原(BCMA)和 CD3 的双特异性抗体(BsAbs),为多发性骨髓瘤(MM)患者的 BsAb 给药提供了一种很有前途的替代方法。在本研究中,我们使用异种移植模型评估了这种方法的疗效。与载体对照组相比,用编码 BCMA/CD3 BsAb 的质粒 DNA 进行单次电穿孔增强肌内注射治疗的小鼠,肿瘤生长明显延迟。治疗后观察到有限的毒性。这项研究表明,用于递送 BCMA/CD3 BsAb 的基于基因治疗的方法对于 MM 的治疗是有效且安全的。