The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors

Abstract Malignant peripheral nerve sheath tumor (MPNST) is a highly aggressive sarcoma that may be seen in patients with neurofibromatosis type 1 (NF1) or occur sporadically. While surgery is the primary treatment for localized MPNST with a 61.9% overall survival rate, metastatic disease is often f...

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Main Authors: Imad Soukar, Robert J. Fisher, Sanjana Bhagavatula, Marianne Collard, Philip A. Cole, Rhoda M. Alani
Format: Article
Language:English
Published: Nature Portfolio 2025-03-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-94517-w
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author Imad Soukar
Robert J. Fisher
Sanjana Bhagavatula
Marianne Collard
Philip A. Cole
Rhoda M. Alani
author_facet Imad Soukar
Robert J. Fisher
Sanjana Bhagavatula
Marianne Collard
Philip A. Cole
Rhoda M. Alani
author_sort Imad Soukar
collection DOAJ
description Abstract Malignant peripheral nerve sheath tumor (MPNST) is a highly aggressive sarcoma that may be seen in patients with neurofibromatosis type 1 (NF1) or occur sporadically. While surgery is the primary treatment for localized MPNST with a 61.9% overall survival rate, metastatic disease is often fatal due to resistance to systemic therapies which underscores the urgent need for effective treatments. MPNSTs frequently harbor inactivating driver mutations in the PRC2 epigenetic repressor complex suggesting epigenetic therapies may represent a specific vulnerability in these tumors. Here, we investigate the role of the LSD1-HDAC1-CoREST (LHC) repressor complex in mediating MPNST tumor growth and progression. Our findings demonstrate that the LHC small molecule inhibitor, corin, induces apoptosis and significantly inhibits proliferation in MPNST cells. Transcriptomic analysis of corin-treated MPNST cells demonstrates specific increases in genes associated with axonogenesis and neuronal differentiation as well as altered extracellular matrix; additionally, corin treatment is shown to inhibit MPNST invasion in vitro. These results underscore the critical role of the LHC complex in facilitating MPNST growth and progression and suggest that targeting the LHC complex represents a promising therapeutic approach for this aggressive malignancy.
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spelling doaj-art-84bcb9c0ee4f4a0f901ecda0e8e10a892025-08-20T03:40:44ZengNature PortfolioScientific Reports2045-23222025-03-0115111210.1038/s41598-025-94517-wThe CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumorsImad Soukar0Robert J. Fisher1Sanjana Bhagavatula2Marianne Collard3Philip A. Cole4Rhoda M. Alani5Department of Dermatology, Boston University Chobanian and Avedisian School of MedicineDepartment of Dermatology, Boston University Chobanian and Avedisian School of MedicineDepartment of Dermatology, Boston University Chobanian and Avedisian School of MedicineDepartment of Dermatology, Boston University Chobanian and Avedisian School of MedicineDivision of Genetics, Departments of Medicine and Biological Chemistry and Molecular Pharmacology, Harvard Medical School and Brigham and Women’s HospitalDepartment of Dermatology, Boston University Chobanian and Avedisian School of MedicineAbstract Malignant peripheral nerve sheath tumor (MPNST) is a highly aggressive sarcoma that may be seen in patients with neurofibromatosis type 1 (NF1) or occur sporadically. While surgery is the primary treatment for localized MPNST with a 61.9% overall survival rate, metastatic disease is often fatal due to resistance to systemic therapies which underscores the urgent need for effective treatments. MPNSTs frequently harbor inactivating driver mutations in the PRC2 epigenetic repressor complex suggesting epigenetic therapies may represent a specific vulnerability in these tumors. Here, we investigate the role of the LSD1-HDAC1-CoREST (LHC) repressor complex in mediating MPNST tumor growth and progression. Our findings demonstrate that the LHC small molecule inhibitor, corin, induces apoptosis and significantly inhibits proliferation in MPNST cells. Transcriptomic analysis of corin-treated MPNST cells demonstrates specific increases in genes associated with axonogenesis and neuronal differentiation as well as altered extracellular matrix; additionally, corin treatment is shown to inhibit MPNST invasion in vitro. These results underscore the critical role of the LHC complex in facilitating MPNST growth and progression and suggest that targeting the LHC complex represents a promising therapeutic approach for this aggressive malignancy.https://doi.org/10.1038/s41598-025-94517-wCoRESTCorinHDACLSD1MPNSTEpigenetics
spellingShingle Imad Soukar
Robert J. Fisher
Sanjana Bhagavatula
Marianne Collard
Philip A. Cole
Rhoda M. Alani
The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
Scientific Reports
CoREST
Corin
HDAC
LSD1
MPNST
Epigenetics
title The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
title_full The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
title_fullStr The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
title_full_unstemmed The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
title_short The CoREST complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
title_sort corest complex is a therapeutic vulnerability in malignant peripheral nerve sheath tumors
topic CoREST
Corin
HDAC
LSD1
MPNST
Epigenetics
url https://doi.org/10.1038/s41598-025-94517-w
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