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新型 FLT3 和 MERTK 双重抑制剂 MRX-2843 与 venetoclax 联合应用对 FLT3-ITD AML 具有有前景的抗白血病活性。

Combining the novel FLT3 and MERTK dual inhibitor MRX-2843 with venetoclax results in promising antileukemic activity against FLT3-ITD AML.

机构信息

Department of Pediatric Hematology, The First Hospital of Jilin University, Changchun, PR China.

National Engineering Laboratory for AIDS Vaccine, Key Laboratory for Molecular Enzymology and Engineering, the Ministry of Education, School of Life Sciences, Jilin University, Changchun, PR China.

出版信息

Leuk Res. 2024 Sep;144:107547. doi: 10.1016/j.leukres.2024.107547. Epub 2024 Jun 24.

Abstract

FMS-like tyrosine kinase 3 (FLT3) mutations occur in approximately one third of acute myeloid leukemia (AML) patients. FLT3-Internal tandem duplication (FLT3-ITD) mutations are the most common FLT3 mutations and are associated with a poor prognosis. Gilteritinib is a FLT3 inhibitor that is US FDA approved for treating adult patients with relapsed/refractory AML and a FLT3 mutation. While gilteritinib monotherapy has improved patient outcome, few patients achieve durable responses. Combining gilteritinib with venetoclax (VEN) appears to make further improvements, though early results suggest that patients with prior exposure to VEN fair much worse than those without prior exposure. MRX-2843 is a promising inhibitor of FLT3 and MERTK. We recently demonstrated that MRX-2843 is equally potent as gilteritinib in FLT3-ITD AML cell lines in vitro and primary patient samples ex vivo. In this study, we investigated the combination of VEN and MRX-2843 against FLT3-ITD AML cells. We found that VEN synergistically enhances cell death induced by MRX-2843 in FLT3-mutated AML cell lines and primary patient samples. Importantly, we found that VEN synergistically enhances cell death induced by MRX-2843 in FLT3-ITD AML cells with acquired resistance to cytarabine (AraC) or VEN+AraC. VEN and MRX-2843 significantly reduce colony-forming capacity of FLT3-ITD primary AML cells. Mechanistic studies show that MRX-2843 decreases Mcl-1 and c-Myc protein levels via transcriptional regulation and combined MRX-2843 and VEN significantly decreases oxidative phosphorylation in FLT3-ITD AML cells. Our findings highlight a promising combination therapy against FLT3-ITD AML, supporting further in vitro and in vivo testing.

摘要

FMS 样酪氨酸激酶 3(FLT3)突变发生在大约三分之一的急性髓系白血病(AML)患者中。FLT3 内部串联重复(FLT3-ITD)突变是最常见的 FLT3 突变,与不良预后相关。吉特替尼是一种 FLT3 抑制剂,已获得美国食品和药物管理局(FDA)批准,用于治疗复发/难治性 AML 且存在 FLT3 突变的成年患者。虽然吉特替尼单药治疗改善了患者的预后,但很少有患者获得持久缓解。吉特替尼联合维奈托克(VEN)似乎进一步改善了疗效,但早期结果表明,先前接触过 VEN 的患者比未接触过 VEN 的患者预后差得多。MRX-2843 是一种有前途的 FLT3 和 MERTK 抑制剂。我们最近证明,MRX-2843 在体外 FLT3-ITD AML 细胞系和原代患者样本中与吉特替尼同样有效。在这项研究中,我们研究了 VEN 和 MRX-2843 联合治疗 FLT3-ITD AML 细胞的效果。我们发现,VEN 与 MRX-2843 联合可协同增强 FLT3 突变 AML 细胞系和原代患者样本中由 MRX-2843 诱导的细胞死亡。重要的是,我们发现 VEN 与 MRX-2843 联合可协同增强对阿糖胞苷(AraC)或 VEN+AraC 获得性耐药的 FLT3-ITD AML 细胞的细胞死亡。VEN 和 MRX-2843 可显著降低 FLT3-ITD 原代 AML 细胞的集落形成能力。机制研究表明,MRX-2843 通过转录调控降低 Mcl-1 和 c-Myc 蛋白水平,联合使用 MRX-2843 和 VEN 可显著降低 FLT3-ITD AML 细胞中的氧化磷酸化。我们的研究结果突出了一种针对 FLT3-ITD AML 的很有前途的联合治疗方法,支持进一步的体外和体内研究。

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