Publication Date

1-1-2023

Journal

Frontiers in Oncology

DOI

10.3389/fonc.2023.1229696

PMID

37593097

PMCID

PMC10430779

PubMedCentral® Posted Date

8-1-2023

PubMedCentral® Full Text Version

Post-Print

Published Open-Access

yes

Keywords

mutant p53, Y220C, artificial intelligence, small molecule inhibitors, apoptosis

Abstract

Introduction

The p53-Y220C mutation is one of the most common mutations that play a major role in cancer progression.

Methods

In this study, we applied artificial intelligence (AI)-powered virtual screening to identify small-molecule compounds that specifically restore the wild-type p53 conformation from p53-Y220C. From 10 million compounds, the AI algorithm selected a chemically diverse set of 83 high-scoring hits, which were subjected to several experimental assays using cell lines with different p53 mutations.

Results

We identified one compound, H3, that preferentially killed cells with the p53-Y220C mutation compared to cells with other p53 mutations. H3 increased the amount of folded mutant protein with wild-type p53 conformation, restored its transcriptional functions, and caused cell cycle arrest and apoptosis. Furthermore, H3 reduced tumorigenesis in a mouse xenograft model with p53-Y220C-positive cells.

Conclusion

AI enabled the discovery of the H3 compound that selectively reactivates the p53-Y220C mutant and inhibits tumor development in mice.

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