The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma

Abstract Oral tumors are relatively common in dogs, and canine oral squamous cell carcinoma (COSCC) is the most prevalent oral malignancy of epithelial origin. COSCC is locally aggressive with up to 20% of patients showing regional or distant metastasis at the time of diagnosis. The treatment of cho...

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Main Authors: William P. Katt, Cheryl E. Balkman, Scott D. Butler, Michael Byron, Patrick C. Carney, Amy B. Todd-Donato, Matthew E. Drozd, Gerald E. Duhamel, Jacquelyn M. Evans, Nadine Fiani, Jordan C. Ford, Jennifer K. Grenier, Jessica J. Hayward, Kristiina Heikinheimo, Kelly R. Hume, Elizabeth S. Moore, Rishi Puri, Skylar R. Sylvester, Sydney L. Warshaw, Suzin M. Webb, Andrew C. White, Alexandra L. Wright, Richard A. Cerione, Santiago Peralta
Format: Article
Language:English
Published: Nature Portfolio 2025-02-01
Series:Scientific Reports
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Online Access:https://doi.org/10.1038/s41598-025-90574-3
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author William P. Katt
Cheryl E. Balkman
Scott D. Butler
Michael Byron
Patrick C. Carney
Amy B. Todd-Donato
Matthew E. Drozd
Gerald E. Duhamel
Jacquelyn M. Evans
Nadine Fiani
Jordan C. Ford
Jennifer K. Grenier
Jessica J. Hayward
Kristiina Heikinheimo
Kelly R. Hume
Elizabeth S. Moore
Rishi Puri
Skylar R. Sylvester
Sydney L. Warshaw
Suzin M. Webb
Andrew C. White
Alexandra L. Wright
Richard A. Cerione
Santiago Peralta
author_facet William P. Katt
Cheryl E. Balkman
Scott D. Butler
Michael Byron
Patrick C. Carney
Amy B. Todd-Donato
Matthew E. Drozd
Gerald E. Duhamel
Jacquelyn M. Evans
Nadine Fiani
Jordan C. Ford
Jennifer K. Grenier
Jessica J. Hayward
Kristiina Heikinheimo
Kelly R. Hume
Elizabeth S. Moore
Rishi Puri
Skylar R. Sylvester
Sydney L. Warshaw
Suzin M. Webb
Andrew C. White
Alexandra L. Wright
Richard A. Cerione
Santiago Peralta
author_sort William P. Katt
collection DOAJ
description Abstract Oral tumors are relatively common in dogs, and canine oral squamous cell carcinoma (COSCC) is the most prevalent oral malignancy of epithelial origin. COSCC is locally aggressive with up to 20% of patients showing regional or distant metastasis at the time of diagnosis. The treatment of choice most typically involves wide surgical excision. Although long-term remission is possible, treatments are associated with considerable morbidity and can negatively impact functionality and quality of life. OSCCs have substantial upregulation of the RAS-RAF-MEK-MAPK signaling axis, and we had previously hypothesized that small-molecule inhibitors that target RAS signaling might effectively inhibit tumor growth and progression. Here, we demonstrate that the MEK inhibitor trametinib, an FDA-approved drug for human cancers, substantially inhibits the growth of six COSCC cell lines established from current patient tumor samples. We further show preliminary clinical evidence that the drug is able to cause ~ 40% and ~ 80% tumor regression in two out of four patients with spontaneously occurring COSCC, a partial response according to commonly used RECIST criteria. Given the limited treatment options available and the number of dogs for which standard of care is not acceptable, these preliminary findings provide new hope that more suitable treatment options may soon enter the veterinary clinic.
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spelling doaj-art-4f9065e06f0c403dbfcdf12c73bc500a2025-08-20T03:04:16ZengNature PortfolioScientific Reports2045-23222025-02-0115111910.1038/s41598-025-90574-3The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinomaWilliam P. Katt0Cheryl E. Balkman1Scott D. Butler2Michael Byron3Patrick C. Carney4Amy B. Todd-Donato5Matthew E. Drozd6Gerald E. Duhamel7Jacquelyn M. Evans8Nadine Fiani9Jordan C. Ford10Jennifer K. Grenier11Jessica J. Hayward12Kristiina Heikinheimo13Kelly R. Hume14Elizabeth S. Moore15Rishi Puri16Skylar R. Sylvester17Sydney L. Warshaw18Suzin M. Webb19Andrew C. White20Alexandra L. Wright21Richard A. Cerione22Santiago Peralta23Department of Molecular Medicine, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityGenomics Facility, Institute of Biotechnology, Cornell UniversityGenomics Facility, Institute of Biotechnology, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Oral and Maxillofacial Surgery, Institute of Dentistry, University of Turku and Turku University HospitalDepartment of Clinical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityPrism Veterinary DentistryDepartment of Clinical Sciences, Cornell UniversityDepartment of Biomedical Sciences, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityDepartment of Molecular Medicine, Cornell UniversityDepartment of Clinical Sciences, Cornell UniversityAbstract Oral tumors are relatively common in dogs, and canine oral squamous cell carcinoma (COSCC) is the most prevalent oral malignancy of epithelial origin. COSCC is locally aggressive with up to 20% of patients showing regional or distant metastasis at the time of diagnosis. The treatment of choice most typically involves wide surgical excision. Although long-term remission is possible, treatments are associated with considerable morbidity and can negatively impact functionality and quality of life. OSCCs have substantial upregulation of the RAS-RAF-MEK-MAPK signaling axis, and we had previously hypothesized that small-molecule inhibitors that target RAS signaling might effectively inhibit tumor growth and progression. Here, we demonstrate that the MEK inhibitor trametinib, an FDA-approved drug for human cancers, substantially inhibits the growth of six COSCC cell lines established from current patient tumor samples. We further show preliminary clinical evidence that the drug is able to cause ~ 40% and ~ 80% tumor regression in two out of four patients with spontaneously occurring COSCC, a partial response according to commonly used RECIST criteria. Given the limited treatment options available and the number of dogs for which standard of care is not acceptable, these preliminary findings provide new hope that more suitable treatment options may soon enter the veterinary clinic.https://doi.org/10.1038/s41598-025-90574-3Oral squamous cell carcinomaCanine cancerTrametinibRAS signaling
spellingShingle William P. Katt
Cheryl E. Balkman
Scott D. Butler
Michael Byron
Patrick C. Carney
Amy B. Todd-Donato
Matthew E. Drozd
Gerald E. Duhamel
Jacquelyn M. Evans
Nadine Fiani
Jordan C. Ford
Jennifer K. Grenier
Jessica J. Hayward
Kristiina Heikinheimo
Kelly R. Hume
Elizabeth S. Moore
Rishi Puri
Skylar R. Sylvester
Sydney L. Warshaw
Suzin M. Webb
Andrew C. White
Alexandra L. Wright
Richard A. Cerione
Santiago Peralta
The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
Scientific Reports
Oral squamous cell carcinoma
Canine cancer
Trametinib
RAS signaling
title The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
title_full The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
title_fullStr The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
title_full_unstemmed The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
title_short The MEK inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
title_sort mek inhibitor trametinib is effective in inhibiting the growth of canine oral squamous cell carcinoma
topic Oral squamous cell carcinoma
Canine cancer
Trametinib
RAS signaling
url https://doi.org/10.1038/s41598-025-90574-3
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