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Abstract

Background: Niclosamide (5-Chloro-N-(2-chloro-4-nitrophenyl)-2-hydroxybenzamide exhibits promising potential in cancer treatment through its exogenous mitochondrial uncoupling mechanism. However, it faces challenges due to its limited oral bioavailability, attributed to its hydrophobic nature [1]. Recent studies have highlighted derivatives such as Niclosamide Ethanolamine (NEN: N-(2-Hydroxyethyl)-5-chloro-N-(2-chloro-4-nitrophenyl)-2-hydroxybenzamide) [3][2] and o-alkyl amino-tethered NIC [4] may offer enhanced efficacy and reduced toxicity in cancer therapeutics. However, the Niclosamide Piperazine (NPP: 4-[(5-Chloro-2-hydroxyphenyl)carbamoyl]-N-(2-chloro-4-nitrophenyl)piperazine) and NEN salt derivatives, has not been as extensively explored despite having better bioavailability than Niclosamide.

Method: This research aims to analyze the efficacy of two proposed compounds NPP and NEN o-alkyl amino-tethered compounds (Figure 1) derivatives as effective cancer treatments by synthesizing its different substituents while sustaining its efficacy. The basis of selection criteria of substituent functional groups follow the study by Terada in 1990 [5] showed that following characteristics are required to act as an uncoupler: 1) an acid dissociable group 2) bulky hydrophobic moiety 3) strong electron-withdrawing group [5]. In addition, the findings based on the study by UTRGV that phenol OH of niclosamide serves a critical role in the anti-glioblastoma activity, while removing Cl (5- or 2’-Cl) or NH2 functional group did not impact its efficacy, were utilized in selection of functional groups [4].

Results: Based on the MTT assay, NPP showed an acceptable IC50 value of 1.50 uM and NEN 1.834 uM on the glioblastoma cells, comparatively similar to IC50 value of Niclosamide, while having better bioavailability.

Conclusions: NPP and NEN showed similar efficacy as Niclosamide, while having enhanced bioavailability. NPP and NEN’s derivatives will be explored more for further enhancing its efficacy.

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Therapeutic Potential of Niclosamide Piperazine and Niclosamide Ethanolamine o-Alkyl Tethered Derivatives in Cancer Treatment

Background: Niclosamide (5-Chloro-N-(2-chloro-4-nitrophenyl)-2-hydroxybenzamide exhibits promising potential in cancer treatment through its exogenous mitochondrial uncoupling mechanism. However, it faces challenges due to its limited oral bioavailability, attributed to its hydrophobic nature [1]. Recent studies have highlighted derivatives such as Niclosamide Ethanolamine (NEN: N-(2-Hydroxyethyl)-5-chloro-N-(2-chloro-4-nitrophenyl)-2-hydroxybenzamide) [3][2] and o-alkyl amino-tethered NIC [4] may offer enhanced efficacy and reduced toxicity in cancer therapeutics. However, the Niclosamide Piperazine (NPP: 4-[(5-Chloro-2-hydroxyphenyl)carbamoyl]-N-(2-chloro-4-nitrophenyl)piperazine) and NEN salt derivatives, has not been as extensively explored despite having better bioavailability than Niclosamide.

Method: This research aims to analyze the efficacy of two proposed compounds NPP and NEN o-alkyl amino-tethered compounds (Figure 1) derivatives as effective cancer treatments by synthesizing its different substituents while sustaining its efficacy. The basis of selection criteria of substituent functional groups follow the study by Terada in 1990 [5] showed that following characteristics are required to act as an uncoupler: 1) an acid dissociable group 2) bulky hydrophobic moiety 3) strong electron-withdrawing group [5]. In addition, the findings based on the study by UTRGV that phenol OH of niclosamide serves a critical role in the anti-glioblastoma activity, while removing Cl (5- or 2’-Cl) or NH2 functional group did not impact its efficacy, were utilized in selection of functional groups [4].

Results: Based on the MTT assay, NPP showed an acceptable IC50 value of 1.50 uM and NEN 1.834 uM on the glioblastoma cells, comparatively similar to IC50 value of Niclosamide, while having better bioavailability.

Conclusions: NPP and NEN showed similar efficacy as Niclosamide, while having enhanced bioavailability. NPP and NEN’s derivatives will be explored more for further enhancing its efficacy.

 

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