Efavirenz is a non-nucleoside reverse transcriptase inhibitor (NNRTI). Efavirenz non-competitively inhibits the transcriptional (Ki=2.93nM) and replication (IC95=2.93nM) activity of HIV-1, thereby blocking viral replication. Efavirenz can be used in antiviral and anticancer research[1-4].
In vitro, Efavirenz (23.32–27.89μM) was used to treat triple-negative breast cancer cell lines MCF10AT, MCF10CA1α, and MDA-MB-231 for 4 days. Efavirenz significantly inhibited cell proliferation, reduced cell division, promoted apoptosis and necrosis, and induced a shift in cell morphology toward an epithelial-like phenotype[5]. Efavirenz (1.5μM) in combination with metformin (5 mM) and fluoxetine (0.9μM) was used to treat HCT116 human colon cancer cells for 24–96 hours. Efavirenz significantly increased cellular ROS levels, leading to cell death, decreased mitochondrial membrane potential and mitochondrial electron transport chain complex I and III activities, while increasing the expression of factors related to DNA damage, apoptosis, autophagy, and necroptosis[6].
In vivo, Efavirenz (10mg/kg) was orally administered to CD1 male mice for 36 days. Efavirenz significantly caused body weight loss and induced depression-like and anxiety-like behaviors[7]. Efavirenz (10mg/kg) was orally administered to CD1 male mice for 36 days. Efavirenz significantly led to body weight loss and increased the expression of appetite-related hormones[8].
References:
[1] Costa B, Vale N. Efavirenz: History, Development and Future. Biomolecules. 2022 Dec 31;13(1):88.
[2] Wang PF, Neiner A, Kharasch ED. Efavirenz Metabolism: Influence of Polymorphic CYP2B6 Variants and Stereochemistry. Drug Metab Dispos. 2019 Oct;47(10):1195-1205.
[3] Zhao T, Zhong R, Zhang X, et al. Efavirenz restored NMDA receptor dysfunction and inhibited epileptic seizures in GluN2A/Grin2a mutant mice. Front Neurosci. 2023 Mar 2;17:1086462.
[4] Ali T, Cashion J, Hannaoui S, et al. Treatment with efavirenz extends survival in a Creutzfeldt-Jakob disease model by regulating brain cholesterol metabolism. JCI Insight. 2025 Jun 19;10(14):e190296.
[5] Chiou PT, Ohms S, Board PG, et al. Efavirenz as a potential drug for the treatment of triple-negative breast cancers. Clin Transl Oncol. 2021 Feb;23(2):353-363.
[6] Kang BG, Shende M, Inci G, et al. Combination of metformin/efavirenz/fluoxetine exhibits profound anticancer activity via a cancer cell-specific ROS amplification. Cancer Biol Ther. 2023 Dec 31;24(1):20-32.
[7] Rojas-Osornio SA, Crespo-Ramírez M, Paredes-Cervantes V, et al. Oral Administration of Efavirenz Dysregulates the Tph2 Gene in Brain Serotonergic Areas and Alters Weight and Mood in Mice. Pharmaceuticals (Basel). 2024 Jun 18;17(6):801.
[8] Rojas-Osornio SA, Manuel-Apolinar L, Crespo-Ramírez M, et al. Efavirenz Interacts with Hormones Involved in Appetite and Satiety, Affecting Body Weight in Mice. Int J Mol Sci. 2026 Jan 11;27(2):735.
Efavirenz是一种非核苷逆转录酶抑制剂（NNRTI）。Efavirenz可非竞争性地抑制HIV-1的逆转（Ki=2.93nM）和复制（IC95=2.93nM）活性，从而阻断病毒复制。Efavirenz可用于抗病毒、抗癌症的相关研究[1-4]。
在体外，Efavirenz（23.32–27.89μM）处理三阴性乳腺癌细胞系MCF10AT、MCF10CA1α和MDA-MB-231 4天。Efavirenz显著抑制细胞增殖，减少细胞分裂，促进细胞凋亡和坏死，同时诱导细胞形态向上皮样表型分化[5]。Efavirenz（1.5μM）与metformin（5mM）和fluoxetine（0.9μM）组合处理HCT116人结肠癌细胞24-96小时。Efavirenz显著增加细胞ROS水平，导致细胞死亡，降低线粒体膜电位和线粒体电子传递链复合物I和III活性，同时增加DNA损伤、凋亡、自噬和坏死性凋亡相关因子的表达[6]。
在体内，Efavirenz（10mg/kg）口服给药于CD1雄性小鼠，持续36天。Efavirenz显著导致体重减轻并诱发抑郁样和焦虑样行为[7]。Efavirenz（10mg/kg）口服于CD1雄性小鼠，持续36天。Efavirenz显著导致体重减轻并增加食欲相关激素表达[8]。