Unraveling the Efficacy of AR Antagonists Bearing N-(4-(Benzyloxy)phenyl)piperidine-1-sulfonamide Scaffold in Prostate Cancer Therapy by Targeting LBP Mutations.
Jun 2025· Journal of Medicinal Chemistry· Vol 68, pp. 11962-11978· 2 citations· 27 references
Medicine
TL;DR
LT16 outperformed existing antiandrogens by fully antagonizing clinical AR mutations and effectively suppressing castration- and enzalutamide-resistant LNCaP cells proliferation in vitro and in vivo, positioning it as a promising and innovative therapeutic for advanced PCa.
Abstract
Point mutations in the androgen receptor (AR) are significant drivers of resistance in prostate cancer (PCa), posing a great challenge to the development of effective treatment strategies. Building on our previous discovery of the suboptimal AR antagonist T1-12, we developed LT16, which contains an N-(4-(benzyloxy)phenyl)piperidine-1-sulfonamide scaffold through structural optimization and comprehensive screening against T878A-mutated AR. LT16 outperformed existing antiandrogens by fully antagonizing clinical AR mutations and effectively suppressing castration- and enzalutamide-resistant LNCaP cells proliferation in vitro. Mechanically, LT16 was found to disrupt AR nuclear translocation, hinder AR homodimerization, and suppress transcription of AR-regulated genes by competitive binding to the ligand binding pocket. Further in vivo experiments demonstrated that LT16 significantly reduced both regular- and enzalutamide-resistant LNCaP tumor volume and serum prostate-specific antigen levels in mice. These findings position LT16 as a promising and innovative therapeutic for advanced PCa, particularly in cases where resistance to current therapies is a concern.
Findings support the concept that direct targeting of the intrinsically disordered AR-TAD enables transcriptome-selective pharmacology, providing a mechanistic framework and a novel therapeutic strategy that may complement conventional AR-directed therapies.
In vitro and in vivo findings support the advancement of EMI-1725 as a promising therapeutic candidate for mCRPC, which showed improved biodistribution across organs and displayed less accumulation in the brain compared to enzalutamide, where off-target binding leads to risk of seizure.
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