Table 5 FDA-approved drugs for AML
Drug name (brand name) | Drug type (PubChem CID) | Cancer treatment | Mechanism of action | Route of administration |
|---|---|---|---|---|
Arsenic trioxide (Trisenox) | Small molecule (14888) | APL | Induces apoptosis, promotes cell differentiation, suppresses cell proliferation | Intravenous |
Azacitidine (Onureg, Vidaza) | Small molecule (9444) | AML, myelodysplastic syndromes | Inhibits DNA methyltransferase, resulting in hypomethylation of DNA, and incorporates into RNA, resulting in disassembly of polyribosomes, defective methylation and acceptor function of transfer RNA, and inhibition of protein production | Intravenous or subcutaneous |
Cytarabine (Cytosar) | Small molecule (6253) | AML, meningeal leukemia, ALL, CML | Inhibits nucleic acid synthesis and DNA polymerase, blocking the cell cycle progression from G1 to S phase | Intravenous, subcutaneous or intrathecal |
Daunorubicin hydrochloride (Cerubidine) | Small molecule (62770) | AML, ALL | Intercalates between the DNA base pairs, causing the double-strand unwinding via inhibition of Topo II, resulting in single- and double-strand breaks, thus inhibiting DNA and RNA synthesis | Intravenous |
Dexamethasone (Alba-Dex, Baycadron, Decadrol, Decadron, Dextenza, Decasone, Deronil, Dexameth, Dxevo, Gammacorten, Hemady, Hexadrol, Ozurdex, TaperDex, ZoDex) | Small molecule (5743) | Leukemia, lymphoma, multiple myeloma, mycosis fungoides | As a glucocorticoid agonist, binds to GR and translocates to the nucleus to bind GREs, leading to suppress inflammatory cytokines and inducing apoptosis | Oral, intravenous or intramuscular |
Enasidenib mesylate (Idhifa) | Small molecule (90480031) | AML | Selectively inhibits IDH2 mutants, resulting in reduced production of the oncometabolite D2HG, restoration of cell differentiation and clonal proliferation of myeloid lineage cells | Oral |
Gemtuzumab ozogamicin (Mylotarg) | Monoclonal antibody | AML | As an ADC, binds to CD33 on the surface of leukemia cells and releases a drug (calicheamicin derivative) upon internalization, resulting in site-specific DNA double-strand breaks and cell death | Intravenous |
Gilteritinib fumarate (Xospata) | Small molecule (76970819) | AML | Inhibits the internal tandem tyrosine kinase domain of FLT3 and also inhibits AXL and ALK | Oral |
Glasdegib maleate (Daurismo) | Small molecule (122640033) | AML | Binds to the SMO receptor, inhibiting the Hedgehog signaling pathway | Oral |
Idarubicin hydrochloride (Idamycin) | Small molecule (636362) | AML | Intercalates between the DNA base pairs, causing the double-strand unwinding via inhibition of Topo II, resulting in single- and double-strand breaks, thus inhibiting DNA and RNA synthesis | Oral or intravenous |
Ivosidenib (Tibsovo) | Small molecule (71657455) | AML, CCA, myelodysplastic syndrome | Inhibits IDH1 mutants, resulting in reduced production of the oncometabolite D2HG and promotion of cell differentiation | Oral |
Midostaurin (Rydapt) | Small molecule (9829523) | AML, ASM, MCL, SM-AHN | Inhibits PKCα, VEGFR2, c-Kit, PDGFR, FLT3 tyrosine kinases | Oral |
Mitoxantrone hydrochloride (Novantrone) | Small molecule (51082) | AML, prostate cancer | Intercalates between DNA base pairs, causing double-strand unwinding via inhibition of Topo II, resulting in disruption of DNA synthesis and repair | Intravenous |
Olutasidenib (Rezlidhia) | Small molecule (118955396) | AML | Selectively inhibits IDH1 mutants, resulting in reduced production of the oncometabolite D2HG and promotion of cell differentiation | Oral |
Pemigatinib (Pemazyre) | Small molecule (86705695) | AML, CCA, myeloid or lymphoid cancer | Inhibits FGFR1, FGFR2 and FGFR3 kinase | Intravenous |
Prednisone (Deltasone) | Small molecule (5865) | AML, ALL, CLL, CML, HL, MF, NHL | As a glucocorticoid agonist, binds to GR with less affinity than dexamethasone and translocates to the nucleus to bind GREs, resulting in a milder anti-inflammatory effect | Oral |
Quizartinib dihydrochloride (Vanflyta) | Small molecule (25184035) | AML | Inhibits FLT3 kinase | Oral |
Thioguanine (Tabloid, Lanvis) | Small molecule (2723601) | AML | Utilizes HGPRTase to convert to TGMP, which is further converted by cellular kinases to active TGTP, which incorporates into DNA, leading to DNA strand breaks, cell cycle arrest and apoptosis | Oral |
Venetoclax (Venclexta) | Small molecule (49846579) | AML, CLL, SLL | Selectively inhibits the anti-apoptotic BCL-2 on the surface of mitochondria, leading to apoptosis of cancer cells | Oral |
