• Anna Spreafico and Helen J Mackay.
• Princess Margaret Hospital, Division of Medical Oncology and Hematology , 610 University Avenue, Toronto, Ontario, M5G 2M9 , Canada +1 416 846 2253 ; +1 416 946 6546 ; Helen.Mackay@uhn.ca.
• Expert Opin Investig Drugs. 2013 Nov 1; 22 (11): 1485-93.

IntroductionThe PI3K/AKT/mammalian target of rapamycin (mTOR) pathway plays a critical role in controlling cellular metabolism, proliferation and cell cycle regulation. Constitutive activation of this pathway has been demonstrated in many tumor types. Targeting the PI3K/AKT/mTOR pathway is of increasing therapeutic interest.Areas CoveredRidaforolimus belongs to a class of agents known as the rapalogs, first-generation mTOR inhibitors, which inhibit mTORC1, a part of the mTOR complex. Both oral and intravenous formulations of this agent have been tested in Phase II clinical trials for the treatment of solid tumors and hematologic malignancies. Promising results have been seen in endometrial cancer and soft tissue and bone sarcomas.Expert OpinionThis article summarizes the current clinical and biological data on the use of ridaforolimus emerging from these Phase II studies, and provides potential advantages and drawbacks of the mTOR inhibitors in the current clinical practice.

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