Journal
LANCET HAEMATOLOGY
Volume 8, Issue 12, Pages E922-E933Publisher
ELSEVIER SCI LTD
DOI: 10.1016/S2352-3026(21)00270-2
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Funding
- Charif Souki Cancer Research Grant
- MD Anderson Cancer Center Leukemia specialized program of research excellence [CA100632]
- Cancer Center Support Grant [P30CA016672]
- Leukemia & Lymphoma Society Specialized Center of Research (Strasser)
- Medical Research Future Fund of Australia
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Since 2017, the US FDA has approved nine agents for acute myeloid leukaemia, including Bcl-2 inhibitors, FLT3 inhibitors, and IDH inhibitors. Some of the approved agents are used as single-agent therapies or specific combinations for narrow indications, offering limited treatment value.
Research has resulted in regulatory approval of nine agents for acute myeloid leukaemia indications by the US Food and Drug Administration since 2017: the Bcl-2 inhibitor, venetoclax; two FLT3 inhibitors, midostaurin and gilteritinib; two IDH inhibitors, ivosidenib (IDH1 inhibitor) and enasidenib (IDH2 inhibitor); the anti-CD33 antibody-drug conjugate, gemtuzumab ozogamicin; the oral, poorly absorbable hypomethylating agent, azacitidine; the liposomal formulation of cytarabine and daunorubicin (5:1 ratio), CPX-351; and the hedgehog signalling pathway inhibitor, glasdegib. A 100% absorbable oral formulation of the hypomethylating agent decitabine was approved for the treatment of myelodysplastic syndrome and chronic myelomonocytic leukaemia, and might be used as an alternative to parenteral hypomethylating agents. Several of the approvals are as single-agent therapies or in specific combinations for narrow indications, thus offering poor treatment value. In this Review, we discuss ongoing research into combinations containing these commercially available targeted therapies for acute myeloid leukaemia.
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