Background: Ciclopirox (CPX), a broad-spectrum fungicide, has been widely used to treat fungal infection on the skin and nails for decades. Recent preclinical and clinical studies have shown that CPX also possesses promising anticancer activity.

Objective: The objective of this study is to summarize the patents, the pharmacological and toxicological properties, the anticancer activity, and the mechanisms of action of CPX and its derivatives as anticancer agents.

Methods: PubMed and Google using the keywords “ciclopirox”, “cancer or tumor” and “patent” were searched, and the identified literature was reviewed.

Results: Pharmacological and toxicological profiles from preclinical and clinical studies support that systemic administration of CPX and its derivatives is feasible and safe for cancer treatment. CPX exerts its anticancer activity by inhibiting cell proliferation, inducing apoptosis, suppressing cell migration and invasion, and inhibiting angiogenesis and lymphangiogenesis. Mechanistically, CPX impacts the expression or activities of multiple signaling molecules or pathways, such as ribonucleotide reductase, Myc, DJ-1, Wnt/β-catenin, DOHH/eIF5A/PEAK1, VEGFR-3/ERK1/2, ATR/Chk1/Cdc25A, and AMPK/TSC/mTORC1. Most of these effects are attributed to iron chelation by CPX. Five patents have been retrieved: four patents on the development of CPX prodrugs to improve the water solubility and bioavailability of CPX, and one patent on the methods of bladder cancer treatment with CPX, CPX-O, or a CPX prodrug.

Conclusion: CPX has a great potential to be repositioned for cancer therapy.

背景：环吡酮 (CPX) 是一种广谱杀菌剂，几十年来一直被广泛用于治疗皮肤和指甲的真菌感染。最近的临床前和临床研究表明，CPX 还具有良好的抗癌活性。

目的：本研究的目的是总结CPX及其衍生物作为抗癌剂的专利、药理毒理特性、抗癌活性及作用机制。

方法：使用关键词“环吡酮”、“癌症或肿瘤”和“专利”对 PubMed 和 Google 进行搜索，并对已识别的文献进行回顾。

结果：临床前和临床研究的药理学和毒理学特征支持 CPX 及其衍生物的全身给药对于癌症治疗是可行和安全的。CPX通过抑制细胞增殖、诱导细胞凋亡、抑制细胞迁移和侵袭、抑制血管生成和淋巴管生成来发挥其抗癌活性。从机制上讲，CPX 影响多种信号分子或通路的表达或活性，例如核糖核苷酸还原酶、Myc、DJ-1、Wnt/β-catenin、DOHH/eIF5A/PEAK1、VEGFR-3/ERK1/2、ATR/Chk1/ Cdc25A 和 AMPK/TSC/mTORC1。大多数这些影响归因于 CPX 的铁螯合。已检索专利五项：四项关于开发CPX前药以提高CPX水溶性和生物利用度的专利，

结论：CPX 在癌症治疗中具有重新定位的巨大潜力。