靶向 CD123嵌合抗原受体T 细胞治疗急性髓系白血病最新进展

摘要

急性髓系白血病（acute myeloid leukemia，AML）是最常见的、死亡率最高的一类白血病。在过去的40年里，尽管人们对于AML的认识更加深入，但是与其他血液肿瘤相比， AML的治疗并没有明显的变化。 CD123是AML相关抗原，高表达于白血病干细胞，低或不表达于正常造血干细胞。作为治疗AML的潜在治疗靶点，近期靶向CD123＋细胞免疫治疗技术已经有了新的进展，尤其是嵌合抗原受体（ chimeric antigen receptor ，CAR） T细胞治疗技术发展迅速。早期研究也已经证实CD123 CAR－T细胞具有抗白血病效应，而且对正常造血系统有不同程度的影响，这也为临床治疗复发／难治AML提供了新的思路。%Acute myeloid leukemia ( AML) is the most common acute leukemia in adults and has the highest death rate of all leukemias. Compared with other hematologic malignancies , there was only a small increment in the 5-year relative overall survival for patients with AML in the last 40 years, despite the advancement in our understanding of AML .CD123 is an AML-associated antigen that expresses at a high level in leukemic stem cells and leukemic blasts and a low level in normal hematopoiet-ic stem cells.As an attractive surface target for AML therapies , immune-based therapies targeting CD 123 are being developed re-cently, especially chimeric antigen receptor ( CAR) T-cell-based immunotherapy .Preclinical data have demonstrated that CD 123 CAR-T cells exhibit potent antileukemic activity and various im-pacts on normal hematopoiesis .This will probably be a promis-ing treatment for patients with relapsed/refractory AML.