Treating metastatic soft-tissue or bone sarcomas - potential role of ridaforolimus

Abstract

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.

Keywords: mTOR; ridaforolimus; sarcoma.

Figure 1

Simplified schematic representation of the mammalian target of rapamycin (mTOR) signaling pathway. Notes: Red, pathway inhibitor; Green, pathway activator; Blue, mTOR complex protein. Abbreviation: NOS, not otherwise specified.