LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma

Abstract Ewing Sarcoma (EwS) is a rare pediatric malignancy characterized by a unique t(11:22) (q24;q12) translocation resulting in the pathognomonic EWSR1::FLI1 fusion. Recent reports indicate that the EWSR1::FLI1 oncofusion drives aberrant expression of numerous transcripts, including Lipoxygenase...

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Main Authors: Tatiana Blanchard, Pouya Faridi, Chong Xu, Adham S. Bear, Reyaz ur Rasool, Grace Huang, Terry C. C. Lim, Rochelle Ayala, Khatuna Gabunia, Mei Ji, Avery D. Posey, John Scholler, Irfan A. Asangani, Anthony W. Purcell, Gerald P. Linette, Carl H. June, Beatriz M. Carreno
Format: Article
Language:English
Published: Nature Portfolio 2025-04-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-025-96877-9
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author Tatiana Blanchard
Pouya Faridi
Chong Xu
Adham S. Bear
Reyaz ur Rasool
Grace Huang
Terry C. C. Lim
Rochelle Ayala
Khatuna Gabunia
Mei Ji
Avery D. Posey
John Scholler
Irfan A. Asangani
Anthony W. Purcell
Gerald P. Linette
Carl H. June
Beatriz M. Carreno
author_facet Tatiana Blanchard
Pouya Faridi
Chong Xu
Adham S. Bear
Reyaz ur Rasool
Grace Huang
Terry C. C. Lim
Rochelle Ayala
Khatuna Gabunia
Mei Ji
Avery D. Posey
John Scholler
Irfan A. Asangani
Anthony W. Purcell
Gerald P. Linette
Carl H. June
Beatriz M. Carreno
author_sort Tatiana Blanchard
collection DOAJ
description Abstract Ewing Sarcoma (EwS) is a rare pediatric malignancy characterized by a unique t(11:22) (q24;q12) translocation resulting in the pathognomonic EWSR1::FLI1 fusion. Recent reports indicate that the EWSR1::FLI1 oncofusion drives aberrant expression of numerous transcripts, including Lipoxygenase Homology Domains 1 (LOXHD1). Given its highly restricted protein expression pattern and role in EwS tumorigenesis and metastasis, LOXHD1 may serve as a novel immunotherapeutic target in this malignancy. LOXHD1 immunogenic epitopes restricted to HLA-A*02:01 allowed for the isolation of a high avidity αβTCR. LOXHD1-specific TCR engineered CD8+ T cells conferred cytotoxic activity against a panel of HLA-A*02:01+ EwS tumor cell lines and adoptive transfer led to tumor eradication in a mouse xenograft model of EwS. This study nominates LOXHD1 as an oncofusion regulated, non-mutated tumor associated antigen (TAA) with expression limited to inner hair cells of the cochlea, adult testis, and EwS.
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spelling doaj-art-71ede091145a4392a1a07eed8a28b41d2025-08-20T02:17:47ZengNature PortfolioScientific Reports2045-23222025-04-0115111310.1038/s41598-025-96877-9LOXHD1 is an oncofusion-regulated antigen of ewing sarcomaTatiana Blanchard0Pouya Faridi1Chong Xu2Adham S. Bear3Reyaz ur Rasool4Grace Huang5Terry C. C. Lim6Rochelle Ayala7Khatuna Gabunia8Mei Ji9Avery D. Posey10John Scholler11Irfan A. Asangani12Anthony W. Purcell13Gerald P. Linette14Carl H. June15Beatriz M. Carreno16Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of PennsylvaniaCentre for Cancer Research, Hudson Institute of Medical Research, Monash UniversityCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaDivision of Hematology-Oncology, Department of Medicine, Perelman School of Medicine, University of PennsylvaniaDepartment of Cancer Biology, Perelman School of Medicine, University of Pennsylvania, BRBII/IIICentre for Cancer Research, Hudson Institute of Medical Research, Monash UniversityCentre for Cancer Research, Hudson Institute of Medical Research, Monash UniversityDepartment of Biochemistry and Molecular Biology and Infection and Immunity Program, Biomedicine Discovery Institute, Monash UniversityCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaDepartment of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of PennsylvaniaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaDepartment of Cancer Biology, Perelman School of Medicine, University of Pennsylvania, BRBII/IIIDepartment of Biochemistry and Molecular Biology and Infection and Immunity Program, Biomedicine Discovery Institute, Monash UniversityCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaCenter for Cellular Immunotherapies, Perelman School of Medicine, University of PennsylvaniaAbstract Ewing Sarcoma (EwS) is a rare pediatric malignancy characterized by a unique t(11:22) (q24;q12) translocation resulting in the pathognomonic EWSR1::FLI1 fusion. Recent reports indicate that the EWSR1::FLI1 oncofusion drives aberrant expression of numerous transcripts, including Lipoxygenase Homology Domains 1 (LOXHD1). Given its highly restricted protein expression pattern and role in EwS tumorigenesis and metastasis, LOXHD1 may serve as a novel immunotherapeutic target in this malignancy. LOXHD1 immunogenic epitopes restricted to HLA-A*02:01 allowed for the isolation of a high avidity αβTCR. LOXHD1-specific TCR engineered CD8+ T cells conferred cytotoxic activity against a panel of HLA-A*02:01+ EwS tumor cell lines and adoptive transfer led to tumor eradication in a mouse xenograft model of EwS. This study nominates LOXHD1 as an oncofusion regulated, non-mutated tumor associated antigen (TAA) with expression limited to inner hair cells of the cochlea, adult testis, and EwS.https://doi.org/10.1038/s41598-025-96877-9
spellingShingle Tatiana Blanchard
Pouya Faridi
Chong Xu
Adham S. Bear
Reyaz ur Rasool
Grace Huang
Terry C. C. Lim
Rochelle Ayala
Khatuna Gabunia
Mei Ji
Avery D. Posey
John Scholler
Irfan A. Asangani
Anthony W. Purcell
Gerald P. Linette
Carl H. June
Beatriz M. Carreno
LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma
Scientific Reports
title LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma
title_full LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma
title_fullStr LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma
title_full_unstemmed LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma
title_short LOXHD1 is an oncofusion-regulated antigen of ewing sarcoma
title_sort loxhd1 is an oncofusion regulated antigen of ewing sarcoma
url https://doi.org/10.1038/s41598-025-96877-9
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