Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.

Pancreatic ductal adenocarcinoma (PDAC) is a fatal malignancy. Personalized medicine based on genetic mutations is required to improve its prognosis. The PI3K/AKT pathway plays a crucial role in cancer progression. While PI3K inhibitors have been developed for several malignancies, none have been cl...

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Main Authors: Yoshimasa Suzuki, Makoto Sugimori, Yushi Kanemaru, Sho Onodera, Hiromi Tsuchiya, Aya Ikeda, Ryosuke Ikeda, Yoshihiro Goda, Hiroaki Kaneko, Kuniyasu Irie, Soichiro Sue, Hideaki Ijichi, Shin Maeda
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2025-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0326491
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author Yoshimasa Suzuki
Makoto Sugimori
Yushi Kanemaru
Sho Onodera
Hiromi Tsuchiya
Aya Ikeda
Ryosuke Ikeda
Yoshihiro Goda
Hiroaki Kaneko
Kuniyasu Irie
Soichiro Sue
Hideaki Ijichi
Shin Maeda
author_facet Yoshimasa Suzuki
Makoto Sugimori
Yushi Kanemaru
Sho Onodera
Hiromi Tsuchiya
Aya Ikeda
Ryosuke Ikeda
Yoshihiro Goda
Hiroaki Kaneko
Kuniyasu Irie
Soichiro Sue
Hideaki Ijichi
Shin Maeda
author_sort Yoshimasa Suzuki
collection DOAJ
description Pancreatic ductal adenocarcinoma (PDAC) is a fatal malignancy. Personalized medicine based on genetic mutations is required to improve its prognosis. The PI3K/AKT pathway plays a crucial role in cancer progression. While PI3K inhibitors have been developed for several malignancies, none have been clinically applied to PDAC. PIK3CA encodes the catalytic subunit of Class IA PI3K, and an activating mutation such as E545K and H1047R is oncogenic. In this study, we developed a novel pancreatic cancer mouse model with PIK3CAH1047R mutation, designated Ptf1acre/+; Rosa26-LSL-PIK3CAH1047R:p53loxP/loxP (PPC) mice. At 150 days of age, PPC mice developed PDAC and AKT was activated in their tumor epithelial cells. We established a pancreatic cancer cell line from PPC mice, and alpelisib, an inhibitor of PI3K p110α, inhibited the proliferation of PPC cells in vitro. Furthermore, PPC cells were subcutaneously transplanted into NOD/SCID mice, and alpelisib significantly reduced the tumor burden of PPC cells. Western blotting upon treatment with alpelisib revealed compensatory activation of ERK in PPC cells. Combination treatment with alpelisib and the MEK inhibitor PD98059 significantly inhibited cell proliferation. These data indicate that PIK3CA mutation may be oncogenic in PDAC and that PI3K inhibitors can be effective against such tumors. Dual inhibition of the PI3K/AKT and MEK/ERK pathways may enhance therapeutic effects in PI3K/AKT-activated pancreatic tumors.
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spelling doaj-art-d33f64c01079432c88e458452e98ecaf2025-08-20T03:27:57ZengPublic Library of Science (PLoS)PLoS ONE1932-62032025-01-01207e032649110.1371/journal.pone.0326491Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.Yoshimasa SuzukiMakoto SugimoriYushi KanemaruSho OnoderaHiromi TsuchiyaAya IkedaRyosuke IkedaYoshihiro GodaHiroaki KanekoKuniyasu IrieSoichiro SueHideaki IjichiShin MaedaPancreatic ductal adenocarcinoma (PDAC) is a fatal malignancy. Personalized medicine based on genetic mutations is required to improve its prognosis. The PI3K/AKT pathway plays a crucial role in cancer progression. While PI3K inhibitors have been developed for several malignancies, none have been clinically applied to PDAC. PIK3CA encodes the catalytic subunit of Class IA PI3K, and an activating mutation such as E545K and H1047R is oncogenic. In this study, we developed a novel pancreatic cancer mouse model with PIK3CAH1047R mutation, designated Ptf1acre/+; Rosa26-LSL-PIK3CAH1047R:p53loxP/loxP (PPC) mice. At 150 days of age, PPC mice developed PDAC and AKT was activated in their tumor epithelial cells. We established a pancreatic cancer cell line from PPC mice, and alpelisib, an inhibitor of PI3K p110α, inhibited the proliferation of PPC cells in vitro. Furthermore, PPC cells were subcutaneously transplanted into NOD/SCID mice, and alpelisib significantly reduced the tumor burden of PPC cells. Western blotting upon treatment with alpelisib revealed compensatory activation of ERK in PPC cells. Combination treatment with alpelisib and the MEK inhibitor PD98059 significantly inhibited cell proliferation. These data indicate that PIK3CA mutation may be oncogenic in PDAC and that PI3K inhibitors can be effective against such tumors. Dual inhibition of the PI3K/AKT and MEK/ERK pathways may enhance therapeutic effects in PI3K/AKT-activated pancreatic tumors.https://doi.org/10.1371/journal.pone.0326491
spellingShingle Yoshimasa Suzuki
Makoto Sugimori
Yushi Kanemaru
Sho Onodera
Hiromi Tsuchiya
Aya Ikeda
Ryosuke Ikeda
Yoshihiro Goda
Hiroaki Kaneko
Kuniyasu Irie
Soichiro Sue
Hideaki Ijichi
Shin Maeda
Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.
PLoS ONE
title Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.
title_full Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.
title_fullStr Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.
title_full_unstemmed Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.
title_short Development of a novel PIK3CA-mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a PI3K inhibitor.
title_sort development of a novel pik3ca mutated pancreatic tumor mouse model and evaluation of the therapeutic effects of a pi3k inhibitor
url https://doi.org/10.1371/journal.pone.0326491
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