Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids

Abstract FMS-like tyrosine kinase 3–internal tandem duplication (FLT3/ITD) is a common driver mutation that presents with a high leukemic burden and its impact on metabolic homeostasis remains to be further investigated. Here, we revealed that the oncogenic activation of FLT3/ITD induced upregulatio...

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Main Authors: Feng Yin, Jing Yang, Hao Luo, Tiantian Yu, Wenhua Lu, Mingyue Zhao, Hongli Du, Shijun Wen, Peng Huang, Yumin Hu
Format: Article
Language:English
Published: Nature Publishing Group 2025-04-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-025-07661-6
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author Feng Yin
Jing Yang
Hao Luo
Tiantian Yu
Wenhua Lu
Mingyue Zhao
Hongli Du
Shijun Wen
Peng Huang
Yumin Hu
author_facet Feng Yin
Jing Yang
Hao Luo
Tiantian Yu
Wenhua Lu
Mingyue Zhao
Hongli Du
Shijun Wen
Peng Huang
Yumin Hu
author_sort Feng Yin
collection DOAJ
description Abstract FMS-like tyrosine kinase 3–internal tandem duplication (FLT3/ITD) is a common driver mutation that presents with a high leukemic burden and its impact on metabolic homeostasis remains to be further investigated. Here, we revealed that the oncogenic activation of FLT3/ITD induced upregulation of target genes of sterol regulatory element-binding proteins (SREBPs) in vivo and in acute myeloid leukemia patients. Quizartinib is a second-generation FLT3 inhibitor that selectively inhibits the activating FLT3 mutations. We demonstrated the critical role of SREBP1 degradation in conferring the response of FLT3/ITD cells to quizartinib. Mechanistically, quizartinib facilitated degradation of the precursor form of SREBP1 via the FLT3/AKT/GSK3 axis and reduced protein levels of its target gene fatty acid synthase (FASN). Lipidomics analysis by Liquid Chromatography Mass Spectrometry (LC-MS) demonstrated that inhibition of FLT3 altered global levels of phospholipids including reduction of cardiolipin, leading to subsequent loss of mitochondrial membrane potential. Pharmacological inhibition of SREBP1 or FASN sensitized FLT3/ITD leukemia cells to quizartinib. Quizartinib combined with SREBP inhibitor fatostatin or FASN inhibitor orlistat provided substantial therapeutic benefit over monotherapies in the murine FLT3/ITD leukemia model. Our results indicated the mechanistic link between FLT3/ITD and SREBP degradation and suggested the combination therapy via targeting FLT3/SREBP/FASN axis.
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spelling doaj-art-e1a2eecd969e4d25901abdaf1890d05d2025-08-20T02:19:57ZengNature Publishing GroupCell Death and Disease2041-48892025-04-0116111410.1038/s41419-025-07661-6Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipidsFeng Yin0Jing Yang1Hao Luo2Tiantian Yu3Wenhua Lu4Mingyue Zhao5Hongli Du6Shijun Wen7Peng Huang8Yumin Hu9State Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterSchool of Biology and Biological Engineering, South China University of TechnologyState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterState Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Cancer Metabolism and Intervention Research Center, Sun Yat-sen University Cancer CenterAbstract FMS-like tyrosine kinase 3–internal tandem duplication (FLT3/ITD) is a common driver mutation that presents with a high leukemic burden and its impact on metabolic homeostasis remains to be further investigated. Here, we revealed that the oncogenic activation of FLT3/ITD induced upregulation of target genes of sterol regulatory element-binding proteins (SREBPs) in vivo and in acute myeloid leukemia patients. Quizartinib is a second-generation FLT3 inhibitor that selectively inhibits the activating FLT3 mutations. We demonstrated the critical role of SREBP1 degradation in conferring the response of FLT3/ITD cells to quizartinib. Mechanistically, quizartinib facilitated degradation of the precursor form of SREBP1 via the FLT3/AKT/GSK3 axis and reduced protein levels of its target gene fatty acid synthase (FASN). Lipidomics analysis by Liquid Chromatography Mass Spectrometry (LC-MS) demonstrated that inhibition of FLT3 altered global levels of phospholipids including reduction of cardiolipin, leading to subsequent loss of mitochondrial membrane potential. Pharmacological inhibition of SREBP1 or FASN sensitized FLT3/ITD leukemia cells to quizartinib. Quizartinib combined with SREBP inhibitor fatostatin or FASN inhibitor orlistat provided substantial therapeutic benefit over monotherapies in the murine FLT3/ITD leukemia model. Our results indicated the mechanistic link between FLT3/ITD and SREBP degradation and suggested the combination therapy via targeting FLT3/SREBP/FASN axis.https://doi.org/10.1038/s41419-025-07661-6
spellingShingle Feng Yin
Jing Yang
Hao Luo
Tiantian Yu
Wenhua Lu
Mingyue Zhao
Hongli Du
Shijun Wen
Peng Huang
Yumin Hu
Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
Cell Death and Disease
title Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
title_full Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
title_fullStr Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
title_full_unstemmed Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
title_short Targeting oncogenic activation of FLT3/SREBP/FASN promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
title_sort targeting oncogenic activation of flt3 srebp fasn promotes the therapeutic effect of quizartinib involving disruption of mitochondrial phospholipids
url https://doi.org/10.1038/s41419-025-07661-6
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