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mTOR inhibitors inhibit the mammalian target of rapamycin (mTOR) by forming a complex with binding proteins and preventing the phosphorylation of mTOR substrates. The inhibition of mTOR leads to cell cycle arrest and inhibits angiogenesis. mTOR inhibitors, such as everolimus, are under investigation for the treatment of AML in combination with other therapies, such as azacitidine.