Drug intelligence / Profile preview

gambogenic acid

Development stage
Preclinical
Modality
Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
01

Overview

Gambogenic acid is a polyprenylated xanthone derivative isolated from the resin of Garcinia hanburyi and is structurally related to gambogic acid[1][4]. It has demonstrated potent antitumor activity in preclinical studies across a range of cancers including lung (notably non-small cell lung cancer), liver, colorectal, breast, gastric, bladder and prostate cancers[4][8]. Mechanistically it acts as an inhibitor of enhancer of zeste homolog 2 (EZH2), specifically binding covalently to Cys668 within the EZH2 SET domain and promoting its degradation via CHIP-mediated ubiquitination; this leads to suppression of H3K27Me3 and reactivation of PRC2-silenced tumor suppressor genes[6]. Gambogenic acid also inhibits fibroblast growth factor receptor (FGFR) signaling pathways—particularly relevant in erlotinib-resistant NSCLC—and exerts its antitumor effects by inducing apoptosis (programmed cell death), ferroptosis (iron-dependent cell death), necroptosis (regulated necrosis), autophagy modulation and cell cycle arrest[4][5][6]. Additionally it impedes tumor invasion/metastasis by downregulating metastasis-related proteins and can enhance chemosensitivity or overcome multidrug resistance in malignancies[4][8][10].

Other names
Gambogenate(Z)-4-[(1S,2S,13S,15R)-7-[(2E)-3,7-dimethylocta-2,6-dienyl]-6,8-dihydroxy-17,17-dimethyl-5-(3-methylbut-2-enyl)-10,14-dioxo-3,16-dioxapentacyclo[11.4.1.02,11.02,15.04,9]octadeca-4,6,8,11-tetraen-15-yl]-2-methylbut-2-enoic acid
02

Targets

EZH2 (Histone-lysine N-methyltransferase EZH2)FGFR1 (Fibroblast growth factor receptor 1)FGFR2 (Keratinocyte growth factor receptor)

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