Lin Ann, Giuliano Christopher J, Sayles Nicole M, Sheltzer Jason M
Cold Spring Harbor Laboratory, Cold Spring Harbor, United States.
Stony Brook University, Stony Brook, United States.
Elife. 2017 Mar 24;6:e24179. doi: 10.7554/eLife.24179.
The Maternal Embryonic Leucine Zipper Kinase (MELK) has been reported to be a genetic dependency in several cancer types. MELK RNAi and small-molecule inhibitors of MELK block the proliferation of various cancer cell lines, and MELK knockdown has been described as particularly effective against the highly-aggressive basal/triple-negative subtype of breast cancer. Based on these preclinical results, the MELK inhibitor OTS167 is currently being tested as a novel chemotherapy agent in several clinical trials. Here, we report that mutagenizing MELK with CRISPR/Cas9 has no effect on the fitness of basal breast cancer cell lines or cell lines from six other cancer types. Cells that harbor null mutations in MELK exhibit wild-type doubling times, cytokinesis, and anchorage-independent growth. Furthermore, MELK-knockout lines remain sensitive to OTS167, suggesting that this drug blocks cell division through an off-target mechanism. In total, our results undermine the rationale for a series of current clinical trials and provide an experimental approach for the use of CRISPR/Cas9 in preclinical target validation that can be broadly applied.
据报道,母源胚胎亮氨酸拉链激酶(MELK)在几种癌症类型中是一种基因依赖性。MELK的RNA干扰和小分子抑制剂可阻断各种癌细胞系的增殖,并且MELK基因敲低已被描述为对侵袭性很强的基底样/三阴性乳腺癌亚型特别有效。基于这些临床前结果,MELK抑制剂OTS167目前正在多项临床试验中作为一种新型化疗药物进行测试。在此,我们报告称,用CRISPR/Cas9诱变MELK对基底样乳腺癌细胞系或其他六种癌症类型的细胞系的适应性没有影响。在MELK中存在无效突变的细胞表现出野生型的倍增时间、胞质分裂和不依赖贴壁的生长。此外,MELK基因敲除细胞系对OTS167仍然敏感,这表明该药物通过脱靶机制阻断细胞分裂。总体而言,我们的结果削弱了当前一系列临床试验的理论依据,并提供了一种可广泛应用于临床前靶点验证的CRISPR/Cas9实验方法。