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. 2025 Aug 26;13:110. doi: 10.1186/s40364-025-00824-9

Table 3.

Summary of targeted therapies and their current developmental status in cancer treatment

Cancer Type Study model Targeted Drug Therapeutic Target Classification Primary Target Combination Strategy Mechanism Research Status Refs.
Colorectal Cancer MC38 STM2457 (METTL3 inhibitor) RNA Modification Modulator METTL3 Monotherapy (pre-treated BM-Mf and BM-MDSCs) Epigenetic modulation In vivo [72]
SW480, HT29; AOM/DSS mouse Nsun2-i4 (NSUN2 inhibitor) RNA Modification Modulator NSUN2 Monotherapy or combination with anti-PD-1 therapy Epigenetic modulation In vitro and in vivo [64]
BALB/c; C57BL/6 mice; xenografts of HCT-116, RKO, and MC38 METTL1-WDR4-IN-1, Shikonin (PKM2 inhibitor) Combination Therapy METTL1, PKM2, CD155 Monotherapy or combination of METTL1-WDR4-IN-1 and Shikonin Epigenetic & metabolic inhibition In vivo [77]
HCT-116/5-FU, SW480/5-FU, SW620/5-FU; xenograft models FX11 (LDHA inhibitor) Metabolic Inhibitor METTL3, LDHA Monotherapy or combination of sh-METTL3 and FX11 Epigenetic & metabolic inhibition In vitro and in vivo [81]
HCT116, DLD1; CRC organoids, HCT116 xenografts, ApcMin/+ mice Sirolimus (Rapamune ®), Temsirolimus (Torisel®, mTORC1 inhibitors) Combination Therapy METTL3, mTORC1 Monotherapy or combination with sh-METTL3 Epigenetic & signaling inhibition In vitro and in vivo [112]
HCT116, DLD1; nude mice xenografts

DAA (pan-methylation

inhibitor)

RNA Modification Modulator METTL3 Monotherapy Epigenetic modulation In vitro and in vivo [86]
Ocular Melanoma 92.1, CRMM1 Oxamate, 2-DG Metabolic Inhibitor Histone lactylation Monotherapy Metabolic inhibition In vitro [70]
OCM1, CRMM1, orthotopic xenograft mouse 2-DG, Oxamate, LDHA/B siRNAs Metabolic Inhibitor Histone lactylation Monotherapy Metabolic inhibition In vitro and in vivo [17]
HCC Huh-7, HepG2 C646 (p300 inhibitor) Lactylation Inhibitor Histone lactylation, p300 Monotherapy Epigenetic modulation In vitro [66]
Post-treatment surgical HCC samples; orthotopic xenograft 2-DG, siIGF2BP3-loaded liposomes (si-LNPs) Combination Therapy IGF2BP3 Combination of lenvatinib with 2-DG or siIGF2BP3-LNPs Epigenetic & Post-translational Regulation In vivo and clinical sample analysis [74]
HepG2, HCC-PDX mouse model Cpd-5 (ALDOA inhibitor) Metabolic Inhibitor ALDOA Monotherapy Metabolic inhibition. In vitro and in vivo [79]
ESCC KYSE30 cell, KYSE30 xenograft Stiripentol (Diacomit®, LDHA inhibitor), Elesclomol (copper ionophore) Lactylation Inhibitor LDHA, NUDT21, FDX1 Combination of Stiripentol with Elesclomol Post-translational Regulation In vitro and in vivo [75]
APL NB4-R1, NB4-R2; BALB/c nude mouse xenograft model GRh2 (histone deacetylase inhibitor) Lactylation Inhibitor Histone lactylation Monotherapy or combination with ATRA Epigenetic modulation In vitro and in vivo [116]
Gastric Cancer HGC-27; xenograft mouse model Elesclomol (cuproptosis inducer), AGK2 (SIRT2 inhibitor) Delactylation Inhibitor METTL16 Combination of elesclomol and AGK2 Post-translational Regulation In vitro and in vivo [73]
LUAD CDX models, PDX models, and KPE mouse model

DAA (pan-methylation

inhibitor), 2-DG, ENOblock (ENO inhibitor)

Combination Therapy m6A, ENO1 Monotherapy Epigenetic & metabolic inhibition In vivo [100]
Leukemia NOMO-1, U937 R-2HG RNA Modification Modulator FTO Monotherapy Metabolic inhibition In vitro [99]
TNBC 4T1 tumor-bearing mice

Remodelin (NAT10

inhibitor)

RNA Modification Modulator NAT10 Monotherapy or combination with anti-CTLA-4 Epigenetic modulation In vivo [111]
ICC HCCC-9810, HuCC-T1; HuCC-T1 allografts in nude mice STM2457 (METTL3 inhibitor) RNA Modification Modulator METTL3 Monotherapy or combination with Gemcitabine Epigenetic modulation In vitro and in vivo [113]
Melanoma B16 cells; B16 mouse ALK-04 (ALKBH5 inhibitor) RNA Modification Modulator ALKBH5 Monotherapy or combination with GVAX and anti-PD-1 Epigenetic modulation In vitro and in vivo [88]