Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line

Abstract Pancreatic ductal adenocarcinoma has a high mortality rate, with a 5-year survival rate of ~ 12%. Therefore, developing new targeted therapies is urgently needed. ONC-201, a promising candidate, is currently undergoing clinical trials. The main objective of the present work is to investigat...

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Main Authors: Zsófia Szász, Angéla Takács, Márton Kalabay, Péter Bárány, Tamás Czuczi, Antal Csámpai, Eszter Lajkó, László Kőhidai
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Language:English
Published: Nature Portfolio 2025-05-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-00070-x
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author Zsófia Szász
Angéla Takács
Márton Kalabay
Péter Bárány
Tamás Czuczi
Antal Csámpai
Eszter Lajkó
László Kőhidai
author_facet Zsófia Szász
Angéla Takács
Márton Kalabay
Péter Bárány
Tamás Czuczi
Antal Csámpai
Eszter Lajkó
László Kőhidai
author_sort Zsófia Szász
collection DOAJ
description Abstract Pancreatic ductal adenocarcinoma has a high mortality rate, with a 5-year survival rate of ~ 12%. Therefore, developing new targeted therapies is urgently needed. ONC-201, a promising candidate, is currently undergoing clinical trials. The main objective of the present work is to investigate the anti-tumour activity of ONC-201 and its two fluorinated analogues (TBP-134, TBP-135). The viability of two pancreatic adenocarcinoma cell lines (PANC-1, MIA PaCa-2) and three other tumour cell lines (A2058, EBC-1, COLO-205) was assessed after 72-hour treatment with drugs at 0.5, 10, and 25 µM. Significant antiproliferative effects were observed, with 0.5 µM TBP-134 achieving the highest potency, reducing cell viability to approximately 50%. None of the molecules exhibited significant cytotoxicity toward normal human dermal fibroblast cells or cardiomyocytes, indicating a selective anti-tumour profile. The analogues showed more effective results than ONC201 on PANC-1 cells (IC50: 0.35 and 1.8 µM vs. IC50: 6.1 µM, respectively). All analogues induced G2/M phase arrest followed by apoptosis in PANC-1 cells. The site of the fluorination influenced the mechanism of apoptotic action of these compounds. Overall, TBP-134 showed superior efficacy, making it a promising candidate for structural optimization within the imipridone family to develop more effective, selective treatments for pancreatic tumours.
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spelling doaj-art-d932ae4a6c9f4fd3878dbde8ed0736d12025-08-20T01:49:43ZengNature PortfolioScientific Reports2045-23222025-05-0115111510.1038/s41598-025-00070-xComparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell lineZsófia Szász0Angéla Takács1Márton Kalabay2Péter Bárány3Tamás Czuczi4Antal Csámpai5Eszter Lajkó6László Kőhidai7Department of Genetics, Cell- and Immunobiology, Semmelweis UniversityDepartment of Genetics, Cell- and Immunobiology, Semmelweis UniversityDepartment of Genetics, Cell- and Immunobiology, Semmelweis UniversityDepartment of Organic Chemistry, Institute of Chemistry, Eötvös Loránd UniversityDepartment of Organic Chemistry, Institute of Chemistry, Eötvös Loránd UniversityDepartment of Organic Chemistry, Institute of Chemistry, Eötvös Loránd UniversityDepartment of Genetics, Cell- and Immunobiology, Semmelweis UniversityDepartment of Genetics, Cell- and Immunobiology, Semmelweis UniversityAbstract Pancreatic ductal adenocarcinoma has a high mortality rate, with a 5-year survival rate of ~ 12%. Therefore, developing new targeted therapies is urgently needed. ONC-201, a promising candidate, is currently undergoing clinical trials. The main objective of the present work is to investigate the anti-tumour activity of ONC-201 and its two fluorinated analogues (TBP-134, TBP-135). The viability of two pancreatic adenocarcinoma cell lines (PANC-1, MIA PaCa-2) and three other tumour cell lines (A2058, EBC-1, COLO-205) was assessed after 72-hour treatment with drugs at 0.5, 10, and 25 µM. Significant antiproliferative effects were observed, with 0.5 µM TBP-134 achieving the highest potency, reducing cell viability to approximately 50%. None of the molecules exhibited significant cytotoxicity toward normal human dermal fibroblast cells or cardiomyocytes, indicating a selective anti-tumour profile. The analogues showed more effective results than ONC201 on PANC-1 cells (IC50: 0.35 and 1.8 µM vs. IC50: 6.1 µM, respectively). All analogues induced G2/M phase arrest followed by apoptosis in PANC-1 cells. The site of the fluorination influenced the mechanism of apoptotic action of these compounds. Overall, TBP-134 showed superior efficacy, making it a promising candidate for structural optimization within the imipridone family to develop more effective, selective treatments for pancreatic tumours.https://doi.org/10.1038/s41598-025-00070-xPancreas adenocarcinomaImipridoneApoptosisONC201Drug developmentTumour selectivity
spellingShingle Zsófia Szász
Angéla Takács
Márton Kalabay
Péter Bárány
Tamás Czuczi
Antal Csámpai
Eszter Lajkó
László Kőhidai
Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line
Scientific Reports
Pancreas adenocarcinoma
Imipridone
Apoptosis
ONC201
Drug development
Tumour selectivity
title Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line
title_full Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line
title_fullStr Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line
title_full_unstemmed Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line
title_short Comparative study of the anti-tumour effects of the imipridone, ONC201 and its fluorinated analogues on pancreatic cancer cell line
title_sort comparative study of the anti tumour effects of the imipridone onc201 and its fluorinated analogues on pancreatic cancer cell line
topic Pancreas adenocarcinoma
Imipridone
Apoptosis
ONC201
Drug development
Tumour selectivity
url https://doi.org/10.1038/s41598-025-00070-x
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