Stein Eytan M, Tallman Martin S
Leukemia Service, Memorial Sloan Kettering Cancer Center, Weill Cornell Medical College, New York, NY.
Blood. 2016 Jan 7;127(1):71-8. doi: 10.1182/blood-2015-07-604538. Epub 2015 Dec 10.
Multiple new drugs are being developed to treat acute myeloid leukemia (AML), including novel formulations of traditional chemotherapy-antibody drug conjugates and agents that target specific mutant enzymes. Next-generation sequencing has allowed us to discover the genetic mutations that lead to the development and clinical progression of AML. Studies of clonal hierarchy suggest which mutations occur early and dominate. This has led to targeted therapy against mutant driver proteins as well as the development of drugs such as CPX-351 and SGN-CD33A whose mechanisms of action and efficacy may not be dependent on mutational complexity. In this brief review, we discuss drugs that may emerge as important for the treatment of AML in the next 10 years.
多种新药正在研发用于治疗急性髓系白血病（AML），包括传统化疗 - 抗体药物偶联物的新型制剂以及靶向特定突变酶的药物。新一代测序技术使我们能够发现导致AML发生和临床进展的基因突变。对克隆层次结构的研究表明哪些突变发生较早并占主导地位。这导致了针对突变驱动蛋白的靶向治疗以及诸如CPX - 351和SGN - CD33A等药物的开发，其作用机制和疗效可能不依赖于突变复杂性。在这篇简短的综述中，我们讨论了未来10年可能对AML治疗具有重要意义的药物。
