Department of Medicine, Division of Medical Oncology, University of Colorado Anschutz Medical Campus, Aurora, Colorado.
Cell Technologies Shared Resources, University of Colorado Anschutz Medical Campus, Aurora, Colorado.
Mol Cancer Ther. 2024 Jan 3;23(1):92-105. doi: 10.1158/1535-7163.MCT-23-0218.
Despite the initial benefit from tyrosine kinase inhibitors (TKI) targeting oncogenic ALK and ROS1 gene fusions in non-small cell lung cancer, complete responses are rare and resistance ultimately emerges from residual tumor cells. Although several acquired resistance mechanisms have been reported at the time of disease progression, adaptative resistance mechanisms that contribute to residual diseases before the outgrowth of tumor cells with acquired resistance are less clear. For the patients who have progressed after TKI treatments, but do not demonstrate ALK/ROS1 kinase mutations, there is a lack of biomarkers to guide effective treatments. Herein, we found that phosphorylation of MIG6, encoded by the ERRFI1 gene, was downregulated by ALK/ROS1 inhibitors as were mRNA levels, thus potentiating EGFR activity to support cell survival as an adaptive resistance mechanism. MIG6 downregulation was sustained following chronic exposure to ALK/ROS1 inhibitors to support the establishment of acquired resistance. A higher ratio of EGFR to MIG6 expression was found in ALK TKI-treated and ALK TKI-resistant tumors and correlated with the poor responsiveness to ALK/ROS1 inhibition in patient-derived cell lines. Furthermore, we identified and validated a MIG6 EGFR-binding domain truncation mutation in mediating resistance to ROS1 inhibitors but sensitivity to EGFR inhibitors. A MIG6 deletion was also found in a patient after progressing to ROS1 inhibition. Collectively, this study identifies MIG6 as a novel regulator for EGFR-mediated adaptive and acquired resistance to ALK/ROS1 inhibitors and suggests EGFR to MIG6 ratios and MIG6-damaging alterations as biomarkers to predict responsiveness to ALK/ROS1 and EGFR inhibitors.
尽管针对非小细胞肺癌中致癌性 ALK 和 ROS1 基因融合的酪氨酸激酶抑制剂 (TKI) 最初具有疗效,但完全缓解罕见,并且耐药性最终还是会由残留肿瘤细胞引起。尽管在疾病进展时已经报道了几种获得性耐药机制,但在获得性耐药肿瘤细胞生长之前,有助于残留疾病的适应性耐药机制还不太清楚。对于 TKI 治疗后进展的患者,但未显示 ALK/ROS1 激酶突变的患者,缺乏生物标志物来指导有效的治疗。在此,我们发现 ERRFI1 基因编码的 MIG6 的磷酸化被 ALK/ROS1 抑制剂下调,同时其 mRNA 水平也下调,从而增强 EGFR 活性以支持细胞存活,作为一种适应性耐药机制。ALK/ROS1 抑制剂慢性暴露后,MIG6 下调得以维持,以支持获得性耐药的建立。在 ALK TKI 治疗和 ALK TKI 耐药肿瘤中发现 EGFR 与 MIG6 的表达比值较高,并且与患者来源细胞系中对 ALK/ROS1 抑制的反应不良相关。此外,我们鉴定并验证了一种 MIG6-EGFR 结合域截断突变,介导对 ROS1 抑制剂的耐药性,但对 EGFR 抑制剂敏感。在对 ROS1 抑制进展的患者中也发现了 MIG6 缺失。总之,这项研究确定了 MIG6 是一种新型调节因子,可介导对 ALK/ROS1 抑制剂的 EGFR 介导的适应性和获得性耐药,并表明 EGFR 与 MIG6 的比值和 MIG6 损伤改变可作为预测对 ALK/ROS1 和 EGFR 抑制剂反应的生物标志物。