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Scaffold repurposing of fendiline: Identification of potent KRAS plasma membrane localization inhibitors
Journal article   Open access   Peer reviewed

Scaffold repurposing of fendiline: Identification of potent KRAS plasma membrane localization inhibitors

Pingyuan Wang, Dharini van der Hoeven, Na Ye, Haiying Chen, Zhiqing Liu, Xiaoping Ma, Dina Montufar-Solis, Kristen M. Rehl, Kwang-Jin Cho, Sabita Thapa, …
European journal of medicinal chemistry, Vol.217, pp.113381-113381
05/05/2021
DOI: 10.1016/j.ejmech.2021.113381
PMCID: PMC12933515
PMID: 33756124
url
https://pmc.ncbi.nlm.nih.gov/articles/PMC12933515/View
Open Access

Abstract

KRAS plays an essential role in regulating cell proliferation, differentiation, migration and survival. Mutated KRAS is a major driver of malignant transformation in multiple human cancers. We showed previously that fendiline (6) is an effective inhibitor of KRAS plasma membrane (PM) localization and function. In this study, we designed, synthesized and evaluated a series of new fendiline analogs to optimize its drug properties. Systemic structure-activity relationship studies by scaffold repurposing led to the discovery of several more active KRAS PM localization inhibitors such as compounds 12f (NY0244), 12h (NY0331) and 22 (NY0335) which exhibit nanomolar potencies. These compounds inhibited oncogenic KRAS-driven cancer cell proliferation at single-digit micromolar concentrations in vitro. In vivo studies in a xenograft model of pancreatic cancer revealed that 12h and 22 suppressed oncogenic KRAS-expressing MiaPaCa-2 tumor growth at a low dose range of 1–5 mg/kg with no vasodilatory effects, indicating their potential as chemical probes and anticancer therapeutics. [Display omitted] •Scaffold repurposing and further structural modifications of fendiline are presented.•Several series of novel diversified analogs have been designed and synthesized.•12f, 12h and 22 are identified as potent and novel KRAS PM localization inhibitors.•In vitro, 12f, 12h and 22 inhibited oncogenic KRAS-driven cancer cell proliferation.•In vivo,12h and 22 suppressed oncogenic KRAS-expressing MiaPaCa-2 tumor growth.
Fendiline KRAS Pancreatic cancer Plasma membrane localization Scaffold repurposing

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