ML327 is a MYC blocker. ML327 can also de-repress E-cadherin transcription and reverse Epithelial-to-Mesenchymal Transition (EMT).

ML327在通过TGF-β1诱导进行上皮-间充质转化（EMT）的NMuMG细胞中部分恢复E-cadherin在质膜的表达。ML327处理在神经母细胞瘤细胞中诱导出延长的形态。用ML327处理的BE(2)-C细胞表现出G1期细胞周期阻滞，伴随S期细胞群体显著减少以及sub G0群体显著增加。ML327在所有七种神经母细胞瘤细胞系中诱导CDH1的表达，CDH1 mRNA表达的诱导范围从50到1,400倍。ML327阻断了在所有测试的神经母细胞瘤细胞系中MYC家族癌基因转录因子的表达。免疫印迹时间进程显示，在用ML327（10 μM）处理2小时内，N-MYC表达早期受到抑制。p53水平也通过用ML327处理而被抑制。

ML327处理导致MYCN表达量下降了两倍，证实了ML327抑制异种移植MYCN表达(p=0.0035)。ML327治疗显著地在两周的治疗期内将肿瘤体积减少了三倍(p=0.02)。在接受ML327治疗的小鼠中，肿瘤移植物的重量大约减轻了三倍(p=0.01)。与接受载体处理的小鼠相比，接受ML327治疗的小鼠体重减轻了12%。

ML327 is solubilized in DMSO for in vitro experiments.Cells are seeded onto 96-well plates at equivalent density (3,000 to 10,000 depending upon cell line), permitted to attach overnight, and treated with either ML327 (10 μM) or vehicle. Daily absorbance measurements (450 nm) using the cell counting kit are obtained. For estimation of IC50 values, cells are plated at equal density, permitted to attach, and baseline absorbance is obtained using cell counting kit. Cells are then treated with varying doses of ML327 (0.1 to 30 μM) and cell viability is measured 72 h after treatment.

ML327 is solubilized in 70% polyethylene glycol for in vivo experiments.Male athymic nude mice (4 to 6 weeks old) are maintained as described. BE(2)-C cells xenografts are established as previously described. 1×106 cells/100 μL of HBSS is injected subcutaneously into flanks using a 26-gauge needle (n=10 per group). Mice are monitored daily for xenograft formation and assessed by measuring the two greatest perpendicular tumor diameter with venier calipers. Xenograft volumes are estimated using the following formula [(length×width2)/2]. Once tumors reach 75 to 100 mm3, mice are randomized to receive either 50 mg/kg of ML327 or control vehicle (70% polyethylene glycol) via intraperitoneal injection twice daily for 14d. Weight and tumor volume are recorded daily. After completion of two weeks of treatment, mice are euthanized and tumors are excised, weighed, and RNA is isolated.