Crose Lisa E S, Linardic Corinne M
Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Sarcoma. 2011;2011:756982. doi: 10.1155/2011/756982. Epub 2011 Jan 2.
Rhabdomyosarcomas (RMSs) are the most common soft tissue sarcomas of childhood and adolescence. To date, there are no effective treatments that target the genetic abnormalities in RMS, and current treatment options for high-risk groups are not adequate. Over the past two decades, research into the molecular mechanisms of RMS has identified key genes and signaling pathways involved in disease pathogenesis. In these studies, members of the receptor tyrosine kinase (RTK) family of cell surface receptors have been characterized as druggable targets for RMS. Through small molecule inhibitors, ligand-neutralizing agents, and monoclonal receptor-blocking antibodies, RTK activity can be manipulated to block oncogenic properties associated with RMS. Herein, we review the members of the RTK family that are implicated in RMS tumorigenesis and discuss both the problems and promise of targeting RTKs in RMS.
横纹肌肉瘤（RMSs）是儿童和青少年中最常见的软组织肉瘤。迄今为止，尚无针对RMS基因异常的有效治疗方法，高危人群目前的治疗选择也并不充分。在过去二十年中，对RMS分子机制的研究已确定了参与疾病发病机制的关键基因和信号通路。在这些研究中，细胞表面受体的受体酪氨酸激酶（RTK）家族成员已被鉴定为RMS的可药物化靶点。通过小分子抑制剂、配体中和剂和单克隆受体阻断抗体，可以操纵RTK活性以阻断与RMS相关的致癌特性。在此，我们综述了与RMS肿瘤发生相关的RTK家族成员，并讨论了在RMS中靶向RTK的问题与前景。
