新的CAR-T治疗方式是否会改变ALL疾病的治疗历史?

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Will novel agents for ALL finally change the natural history?

Pediatric acute lymphoblastic leukemia (ALL) cure rates have markedly improved over the past years to approximately 85%, but remain at 40%-50% in adults. Redefining current adult chemotherapy regimens is likely to improve the natural course of the disease, but new agents are needed. Immunotherapy approaches for pre-B ALL are in the forefront of research on novel agents; in particular, advances are being made in manipulating autologous T cells either by infusion of a bifunctional antibody (eg, blinatumomab) or by ex vivo genetic modification of chimeric antigen receptors (CARs). The natural course of Philadelphia positive ALL has already improved by targeting ABL/BCR1. Other mutated genes are being discovered and novel small molecules that target their products are being studied in clinical trials. Finally, ALL is a heterogeneous disease and novel agents are likely to impact the natural course of smaller populations of biologically defined ALL subtypes.

新的CAR-T治疗方式是否会改变ALL疾病的治疗历史?

儿童急性淋巴细胞白血病（ALL）的治愈率在过去几年有显著改善，约85%，但在成年人中仍保持在40%-50%。当前成人化疗方案可能会提高对该病的自然病程，但仍然需要新的药物来治疗。免疫治疗儿童急性淋巴细胞白血病是新型药物研究的前沿，特别是利用一个双功能抗体（如blinatumomab）或者体内基因修饰的嵌合抗原受体（CARs）操作自体T细胞。 费城的一个试验，通过靶向ABL/BCR1治疗ALL疾病的自然进程有所好转。其他一些变异的基因正在被发现，新型的靶向它们的产品正在临床试验中。最后，ALL疾病是一种血液系统疾病，新型药物很可能会影响所有ALL亚型疾病。

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