1] Department of Orthopaedics, Case Western Reserve University, Cleveland, OH, USA [2] Department of Pathology, Case Western Reserve University, Cleveland, OH, USA.
Oncogenesis. 2012 Nov 19;1(11):e34. doi: 10.1038/oncsis.2012.34.
The survival rate for osteosarcoma patients with localized disease is 70% and only 25% for patients with metastases. Therefore, novel therapeutic and prognostic tools are needed. In this study, extensive screening and validation strategies identified Axl, EphB2, FGFR2, IGF-1R and Ret as specific receptor tyrosine kinases (RTKs) that are activated and promote the in vitro phenotype of two genetically different metastatic osteosarcoma cell lines. Initial phosphoproteomic screening identified twelve RTKs that were phosphorylated in 143B and/or LM7 metastatic human osteosarcoma cells. A small interfering RNA (siRNA) screen demonstrated that siRNA pools targeting ten of the twelve RTKS inhibited the in vitro phenotype of one or both cell lines. To validate the results, we individually tested the four siRNA duplexes that comprised each of the effective siRNA pools from the initial screen. The pattern of phenotype inhibition replicated the pattern of mRNA knockdown by the individual duplexes for seven of the ten RTKs, indicating the effects are consistent with on-target silencing. Five of those seven RTKs were further validated using independent approaches including neutralizing antibodies (IGF-1R), antisense-mediated knockdown (EphB2, FGFR2, and Ret) or small molecule inhibitors (Axl), indicating that those specific RTKs promote the in vitro behavior of metastatic osteosarcoma cell lines and are potential therapeutic targets for osteosarcoma. Immunohistochemistry demonstrated that Axl is frequently activated in osteosarcoma patient biopsy samples, further supporting our screening and validation methods to identify RTKs that may be valuable targets for novel therapies for osteosarcoma patients.
骨肉瘤患者的局部疾病存活率为 70%,而转移患者的存活率仅为 25%。因此,需要新的治疗和预后工具。在这项研究中,广泛的筛选和验证策略确定了 Axl、EphB2、FGFR2、IGF-1R 和 Ret 作为特定的受体酪氨酸激酶(RTKs),它们被激活并促进两种遗传上不同的转移性骨肉瘤细胞系的体外表型。初始磷酸化蛋白质组学筛选确定了 12 个在 143B 和/或 LM7 转移性人骨肉瘤细胞中磷酸化的 RTKs。小干扰 RNA(siRNA)筛选表明,靶向 12 个 RTKs 中的 10 个的 siRNA 池抑制了一个或两个细胞系的体外表型。为了验证结果,我们分别测试了由初始筛选中每个有效 siRNA 池组成的四个 siRNA 双链体。表型抑制的模式复制了十个 RTKs 中七个的 mRNA 敲低的模式,表明这些效果与靶标沉默一致。其中七个 RTKs 中的五个进一步使用独立方法进行了验证,包括中和抗体(IGF-1R)、反义介导的敲低(EphB2、FGFR2 和 Ret)或小分子抑制剂(Axl),表明这些特定的 RTKs 促进了转移性骨肉瘤细胞系的体外行为,并且是骨肉瘤患者新疗法的潜在治疗靶点。免疫组织化学显示 Axl 在骨肉瘤患者活检样本中经常被激活,进一步支持了我们筛选和验证方法,以确定可能对骨肉瘤患者新疗法有价值的 RTKs。