Tomita Akihiro
Department of Hematology and Oncology, Nagoya University Graduate School of Medicine.
Rinsho Ketsueki. 2015 Feb;56(2):130-8. doi: 10.11406/rinketsu.56.130.
Genetic abnormalities including specific point mutations have recently been confirmed by applying comprehensive genome sequencing analyses. Molecular targeting therapies, which focus on the mutated proteins and over-expressed proteins in acute myeloid leukemia (AML) cells, are now being developed in clinical studies and/or based on in vitro analyses. This manuscript summarizes the genetic abnormalities in AML cells and some of the current molecular targeting therapies including FLT3 inhibitors (e.g. AC220; Quizartinib), Polo like kinase 1 (PLK1) inhibitors (e.g. BI-6727; Volasertib), IDH2 inhibitors (e.g. AG-221), and XPO1 inhibitors (e.g. KPT-330; Selinexor).
最近，通过应用全面的基因组测序分析已证实包括特定点突变在内的基因异常。针对急性髓系白血病（AML）细胞中突变蛋白和过表达蛋白的分子靶向疗法目前正在临床研究中和/或基于体外分析进行开发。本文总结了AML细胞中的基因异常以及一些当前的分子靶向疗法，包括FLT3抑制剂（如AC220；Quizartinib）、Polo样激酶1（PLK1）抑制剂（如BI-6727；Volasertib）、IDH2抑制剂（如AG-221）和XPO1抑制剂（如KPT-330；Selinexor）。
