Keedy Vicki L
Vanderbilt University Medical Center, Nashville, TN, USA.
Onco Targets Ther. 2012;5:153-60. doi: 10.2147/OTT.S19055. Epub 2012 Aug 22.
Sarcomas of soft tissue and bone are a rare group of cancers hallmarked by relative insensitivity to cytotoxic chemotherapy. The development of targeted therapies in the treatment of sarcoma has been difficult due to the significant heterogeneity and rarity of these diseases. Inhibition of the mammalian target of rapamycin (mTOR) has emerged as an exciting treatment approach and is being studied extensively in sarcoma patients. Ridaforolimus is a second generation mTOR inhibitor that has shown potential benefit in the treatment of sarcoma. Recently a Phase III study demonstrated an improvement in progression-free survival when patients with at least stable disease after treatment with standard chemotherapy received maintenance ridaforolimus compared to placebo. The results of this study show that mTOR is an important pathway in soft tissue and bone sarcomas and represents an exciting opportunity for the improvement in the treatment of our patients.
软组织和骨肉瘤是一类罕见的癌症,其特征是对细胞毒性化疗相对不敏感。由于这些疾病具有显著的异质性和罕见性,因此在肉瘤治疗中开发靶向疗法一直很困难。抑制哺乳动物雷帕霉素靶蛋白(mTOR)已成为一种令人兴奋的治疗方法,并正在肉瘤患者中进行广泛研究。瑞达法莫司是一种第二代mTOR抑制剂,已显示出在肉瘤治疗中的潜在益处。最近一项III期研究表明,与安慰剂相比,接受标准化疗后至少病情稳定的患者接受瑞达法莫司维持治疗时,无进展生存期有所改善。这项研究的结果表明,mTOR是软组织和骨肉瘤中的一条重要通路,为改善我们患者的治疗提供了一个令人兴奋的机会。