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everolimus + azacitidine is a combination therapy investigated for the treatment of acute myeloid leukemia (AML), particularly in elderly patients or those with relapsed/refractory disease. Everolimus is a small molecule inhibitor of the mechanistic target of rapamycin (mTOR), specifically targeting the mTORC1 complex to inhibit protein synthesis and cell cycle progression. Azacitidine is a hypomethylating agent and pyrimidine nucleoside analog that incorporates into DNA and RNA, leading to the inhibition of DNA methyltransferases (DNMTs). This dual approach aims to overcome resistance mechanisms in AML by simultaneously targeting epigenetic dysregulation and the PI3K/Akt/mTOR signaling pathway. Clinical development for this combination in AML has been primarily led by Bayside Health in Phase 1 trials.
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