Broad Institute of Harvard and Massachusetts Institute of Technology, 7 Cambridge Center, Cambridge, Massachusetts 02142, USA.
Nature. 2010 Dec 16;468(7326):968-72. doi: 10.1038/nature09627. Epub 2010 Nov 24.
Oncogenic mutations in the serine/threonine kinase B-RAF (also known as BRAF) are found in 50-70% of malignant melanomas. Pre-clinical studies have demonstrated that the B-RAF(V600E) mutation predicts a dependency on the mitogen-activated protein kinase (MAPK) signalling cascade in melanoma-an observation that has been validated by the success of RAF and MEK inhibitors in clinical trials. However, clinical responses to targeted anticancer therapeutics are frequently confounded by de novo or acquired resistance. Identification of resistance mechanisms in a manner that elucidates alternative 'druggable' targets may inform effective long-term treatment strategies. Here we expressed ∼600 kinase and kinase-related open reading frames (ORFs) in parallel to interrogate resistance to a selective RAF kinase inhibitor. We identified MAP3K8 (the gene encoding COT/Tpl2) as a MAPK pathway agonist that drives resistance to RAF inhibition in B-RAF(V600E) cell lines. COT activates ERK primarily through MEK-dependent mechanisms that do not require RAF signalling. Moreover, COT expression is associated with de novo resistance in B-RAF(V600E) cultured cell lines and acquired resistance in melanoma cells and tissue obtained from relapsing patients following treatment with MEK or RAF inhibitors. We further identify combinatorial MAPK pathway inhibition or targeting of COT kinase activity as possible therapeutic strategies for reducing MAPK pathway activation in this setting. Together, these results provide new insights into resistance mechanisms involving the MAPK pathway and articulate an integrative approach through which high-throughput functional screens may inform the development of novel therapeutic strategies.
丝氨酸/苏氨酸激酶 B-RAF(也称为 BRAF)中的致癌突变存在于 50-70%的恶性黑色素瘤中。临床前研究表明,B-RAF(V600E)突变预测黑色素瘤对丝裂原活化蛋白激酶 (MAPK) 信号级联的依赖性——这一观察结果已通过 RAF 和 MEK 抑制剂在临床试验中的成功得到验证。然而,针对癌症的靶向治疗的临床反应经常受到新出现或获得性耐药的影响。以阐明替代“可药物”靶标的方式识别耐药机制可能为有效的长期治疗策略提供信息。在这里,我们平行表达了约 600 个激酶和激酶相关的开放阅读框 (ORF),以研究对选择性 RAF 激酶抑制剂的耐药性。我们确定了 MAP3K8(编码 COT/Tpl2 的基因)作为一种 MAPK 途径激动剂,可驱动 B-RAF(V600E)细胞系对 RAF 抑制的耐药性。COT 通过主要依赖 MEK 而不依赖 RAF 信号的机制激活 ERK。此外,COT 表达与 B-RAF(V600E)培养细胞系中的新出现耐药性以及接受 MEK 或 RAF 抑制剂治疗后复发患者的黑色素瘤细胞和组织中的获得性耐药性相关。我们进一步确定组合 MAPK 途径抑制或靶向 COT 激酶活性作为在这种情况下降低 MAPK 途径激活的可能治疗策略。总之,这些结果为涉及 MAPK 途径的耐药机制提供了新的见解,并阐明了一种综合方法,通过该方法,高通量功能筛选可能为新型治疗策略的开发提供信息。
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