尤因肉瘤靶向治疗的最新进展
Recent advances in targeted therapy for Ewing sarcoma.
作者信息
Pishas Kathleen I, Lessnick Stephen L
机构信息
Cancer Therapeutics Laboratory, Center for Personalized Cancer Medicine, Discipline of Medicine, University of Adelaide, Adelaide, SA, Australia; Center for Childhood Cancer and Blood Disorders, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
Center for Childhood Cancer and Blood Disorders, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA; Division of Pediatric Hematology/Oncology/Bone Marrow Transplant, Ohio State University, Columbus, OH, USA.
出版信息
F1000Res. 2016 Aug 25;5. doi: 10.12688/f1000research.8631.1. eCollection 2016.
Ewing sarcoma is an aggressive, poorly differentiated neoplasm of solid bone that disproportionally afflicts the young. Despite intensive multi-modal therapy and valiant efforts, 70% of patients with relapsed and metastatic Ewing sarcoma will succumb to their disease. The persistent failure to improve overall survival for this subset of patients highlights the urgent need for rapid translation of novel therapeutic strategies. As Ewing sarcoma is associated with a paucity of mutations in readily targetable signal transduction pathways, targeting the key genetic aberration and master regulator of Ewing sarcoma, the EWS/ETS fusion, remains an important goal.
尤因肉瘤是一种侵袭性强、分化差的实体骨肿瘤，在年轻人中发病率不成比例地高。尽管进行了强化多模式治疗并付出了巨大努力，但70%复发和转移性尤因肉瘤患者仍会死于该疾病。这部分患者总体生存率持续未能提高，凸显了迅速转化新型治疗策略的迫切需求。由于尤因肉瘤与易于靶向的信号转导通路中突变稀少有关，靶向尤因肉瘤的关键基因畸变和主要调节因子EWS/ETS融合蛋白，仍然是一个重要目标。
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