Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors

Inactivation of p53 due to mutation is observed in approximately half of all human cancer cases, therefore, restoration of the tumor suppressor function of oncogenic p53 mutants represents an attractive and rational therapeutic approach. Currently, multiple mutant p53 reactivators have been develope...

Full description

Saved in:
Bibliographic Details
Main Authors: Kostas A. Papavassiliou, Alice G. Vassiliou, Athanasios G. Papavassiliou
Format: Article
Language:English
Published: Elsevier 2025-09-01
Series:Neoplasia: An International Journal for Oncology Research
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S1476558625000818
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849410022944014336
author Kostas A. Papavassiliou
Alice G. Vassiliou
Athanasios G. Papavassiliou
author_facet Kostas A. Papavassiliou
Alice G. Vassiliou
Athanasios G. Papavassiliou
author_sort Kostas A. Papavassiliou
collection DOAJ
description Inactivation of p53 due to mutation is observed in approximately half of all human cancer cases, therefore, restoration of the tumor suppressor function of oncogenic p53 mutants represents an attractive and rational therapeutic approach. Currently, multiple mutant p53 reactivators have been developed, but only a handful have entered clinical trials [1]. PC14586 (rezatapopt) is an orally available small-molecule reactivator of the Y220C-mutant p53 protein that showed preliminary efficacy and a favorable safety profile in a phase I clinical trial [2] and is now undergoing evaluation in a phase II clinical trial in patients with advanced solid tumors harboring the TP53Y220C mutation (NCT04585750). Although rezatapopt and other p53 mutant reactivators are under clinical assessment, there are still many knowledge gaps that need to be filled to unleash their full therapeutic potential.The preclinical data presented in a recently published research article by Puzio-Kuter et al. achieve to enhance our understanding of the mechanisms and effects of p53-Y220C reactivator compounds and underscore the potential of targeting p53 mutants in cancer therapy [3]. Through this spotlight article, we aim to summarize the findings and emphasize the clinical implications of the study by Puzio-Kutler et al.
format Article
id doaj-art-bf42f53de9404f4caabdcae1703e9f60
institution Kabale University
issn 1476-5586
language English
publishDate 2025-09-01
publisher Elsevier
record_format Article
series Neoplasia: An International Journal for Oncology Research
spelling doaj-art-bf42f53de9404f4caabdcae1703e9f602025-08-20T03:35:18ZengElsevierNeoplasia: An International Journal for Oncology Research1476-55862025-09-016710120110.1016/j.neo.2025.101201Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumorsKostas A. Papavassiliou0Alice G. Vassiliou1Athanasios G. Papavassiliou2First University Department of Respiratory Medicine, ‘Sotiria’ Chest Hospital, Medical School, National and Kapodistrian University of Athens, Athens, GreeceFirst Department of Critical Care Medicine, 'Evangelismos' Hospital, Medical School, National and Kapodistrian University of Athens, Athens, GreeceDepartment of Biological Chemistry, Medical School, National and Kapodistrian University of Athens, Athens, Greece; Corresponding author.Inactivation of p53 due to mutation is observed in approximately half of all human cancer cases, therefore, restoration of the tumor suppressor function of oncogenic p53 mutants represents an attractive and rational therapeutic approach. Currently, multiple mutant p53 reactivators have been developed, but only a handful have entered clinical trials [1]. PC14586 (rezatapopt) is an orally available small-molecule reactivator of the Y220C-mutant p53 protein that showed preliminary efficacy and a favorable safety profile in a phase I clinical trial [2] and is now undergoing evaluation in a phase II clinical trial in patients with advanced solid tumors harboring the TP53Y220C mutation (NCT04585750). Although rezatapopt and other p53 mutant reactivators are under clinical assessment, there are still many knowledge gaps that need to be filled to unleash their full therapeutic potential.The preclinical data presented in a recently published research article by Puzio-Kuter et al. achieve to enhance our understanding of the mechanisms and effects of p53-Y220C reactivator compounds and underscore the potential of targeting p53 mutants in cancer therapy [3]. Through this spotlight article, we aim to summarize the findings and emphasize the clinical implications of the study by Puzio-Kutler et al.http://www.sciencedirect.com/science/article/pii/S1476558625000818Cell-cycle progressionImmune checkpoint inhibitorp53-Y220C reactivatorRezatapoptTumor growth inhibition
spellingShingle Kostas A. Papavassiliou
Alice G. Vassiliou
Athanasios G. Papavassiliou
Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors
Neoplasia: An International Journal for Oncology Research
Cell-cycle progression
Immune checkpoint inhibitor
p53-Y220C reactivator
Rezatapopt
Tumor growth inhibition
title Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors
title_full Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors
title_fullStr Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors
title_full_unstemmed Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors
title_short Rezatapopt: A promising small-molecule “refolder” specific for TP53Y220C mutant tumors
title_sort rezatapopt a promising small molecule refolder specific for tp53y220c mutant tumors
topic Cell-cycle progression
Immune checkpoint inhibitor
p53-Y220C reactivator
Rezatapopt
Tumor growth inhibition
url http://www.sciencedirect.com/science/article/pii/S1476558625000818
work_keys_str_mv AT kostasapapavassiliou rezatapoptapromisingsmallmoleculerefolderspecificfortp53y220cmutanttumors
AT alicegvassiliou rezatapoptapromisingsmallmoleculerefolderspecificfortp53y220cmutanttumors
AT athanasiosgpapavassiliou rezatapoptapromisingsmallmoleculerefolderspecificfortp53y220cmutanttumors