State Key Laboratory of Drug Research, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai, 201203, China.
University of Chinese Academy of Sciences, Beijing, 100049, China.
Acta Pharmacol Sin. 2021 Nov;42(11):1913-1920. doi: 10.1038/s41401-021-00770-w. Epub 2021 Sep 24.
Sepsis is a dysregulated immune response to infection and potentially leads to life-threatening organ dysfunction, which is often seen in serious Covid-19 patients. Disulfiram (DSF), an old drug that has been used to treat alcohol addiction for decades, has recently been identified as a potent inhibitor of the gasdermin D (GSDMD)-induced pore formation that causes pyroptosis and inflammatory cytokine release. Therefore, DSF represents a promising therapeutic for the treatment of inflammatory disorders. Lactoferrin (LF) is a multifunctional glycoprotein with potent antibacterial and anti-inflammatory activities that acts by neutralizing circulating endotoxins and activating cellular responses. In addition, LF has been well exploited as a drug nanocarrier and targeting ligands. In this study, we developed a DSF-LF nanoparticulate system (DSF-LF NP) for combining the immunosuppressive activities of both DSF and LF. DSF-LF NPs could effectively block pyroptosis and inflammatory cytokine release from macrophages. Treatment with DSF-LF NPs showed remarkable therapeutic effects on lipopolysaccharide (LPS)-induced sepsis. In addition, this therapeutic strategy was also applied to treat ulcerative colitis (UC), and substantial treatment efficacy was achieved in a murine colitis model. The underlying mode of action of these DSF-LF-NPs may contribute to efficiently suppressing macrophage-mediated inflammatory responses and ameliorating the complications caused by sepsis and UC. As macrophage pyroptosis plays a pivotal role in inflammation, this safe and effective biomimetic nanomedicine may offer a versatile therapeutic strategy for treating various inflammatory diseases by repurposing DSF.
脓毒症是一种对感染的失调免疫反应,可能导致危及生命的器官功能障碍,这种情况在严重的 COVID-19 患者中经常出现。双硫仑(DSF)是一种已被用于治疗酒精成瘾数十年的老药,最近被确定为一种有效的 Gasdermin D(GSDMD)诱导的孔形成抑制剂,这种孔形成会导致细胞焦亡和炎症细胞因子的释放。因此,DSF 代表了一种有前途的治疗炎症性疾病的方法。乳铁蛋白(LF)是一种具有强大抗菌和抗炎活性的多功能糖蛋白,通过中和循环内毒素和激活细胞反应来发挥作用。此外,LF 已被很好地开发为药物纳米载体和靶向配体。在这项研究中,我们开发了一种 DSF-LF 纳米颗粒系统(DSF-LF NP),将 DSF 和 LF 的免疫抑制活性结合在一起。DSF-LF NPs 可以有效地阻止巨噬细胞中的细胞焦亡和炎症细胞因子的释放。用 DSF-LF NPs 治疗可显著改善脂多糖(LPS)诱导的脓毒症。此外,这种治疗策略也被应用于溃疡性结肠炎(UC)的治疗,在小鼠结肠炎模型中取得了显著的治疗效果。这些 DSF-LF-NPs 的作用机制可能是通过有效抑制巨噬细胞介导的炎症反应,改善脓毒症和 UC 引起的并发症。由于巨噬细胞细胞焦亡在炎症中起着关键作用,这种安全有效的仿生纳米医学可以通过重新利用 DSF 为治疗各种炎症性疾病提供一种多功能的治疗策略。