Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma

Abstract Background Relapsed high‐grade glioma has dismal outcomes. Mebendazole has shown promising activity against glioma in in‐vitro and in‐vivo studies. Hence, we undertook a phase 1 study to repurpose mebendazole in the treatment of glioblastoma. Methods We conducted a phase 1 study (accelerate...

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Main Authors: Vijay M. Patil, Arti Bhelekar, Nandini Menon, Atanu Bhattacharjee, Vijai Simha, Ram Abhinav, Anuja Abhyankar, Epari Sridhar, Abhishek Mahajan, Ameya D. Puranik, Nilendu Purandare, Amit Janu, Ankita Ahuja, Rahul Krishnatry, Tejpal Gupta, Rakesh Jalali
Format: Article
Language:English
Published: Wiley 2020-07-01
Series:Cancer Medicine
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Online Access:https://doi.org/10.1002/cam4.3094
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author Vijay M. Patil
Arti Bhelekar
Nandini Menon
Atanu Bhattacharjee
Vijai Simha
Ram Abhinav
Anuja Abhyankar
Epari Sridhar
Abhishek Mahajan
Ameya D. Puranik
Nilendu Purandare
Amit Janu
Ankita Ahuja
Rahul Krishnatry
Tejpal Gupta
Rakesh Jalali
author_facet Vijay M. Patil
Arti Bhelekar
Nandini Menon
Atanu Bhattacharjee
Vijai Simha
Ram Abhinav
Anuja Abhyankar
Epari Sridhar
Abhishek Mahajan
Ameya D. Puranik
Nilendu Purandare
Amit Janu
Ankita Ahuja
Rahul Krishnatry
Tejpal Gupta
Rakesh Jalali
author_sort Vijay M. Patil
collection DOAJ
description Abstract Background Relapsed high‐grade glioma has dismal outcomes. Mebendazole has shown promising activity against glioma in in‐vitro and in‐vivo studies. Hence, we undertook a phase 1 study to repurpose mebendazole in the treatment of glioblastoma. Methods We conducted a phase 1 study (accelerated titrated design 4) of mebendazole in patients with recurrent glioblastoma (GBM). Patients eligible for re‐irradiation were enrolled in arm A1 (radiation with concurrent temozolomide 75 mg/m2 daily during the course of radiation+mebendazole) while patients who were ineligible were enrolled in either arm B1 (CCNU 110 mg/m2 day 1, every 6 weekly + mebendazole) or arm C1 (temozolomide 200 mg/m2 day 1‐5, every 4 weekly + mebendazole). The primary endpoint of phase 1 was to identify the MTD of mebendazole in each combination. Findings 11 patients were enrolled in the whole study. MTD of mebendazole was not reached in arm A1 and C1 and hence the recommended dose for phase 2 was 1600 mg TDS (4800 mg) per day. The MTD of mebendazole in combination with CCNU was 1600 mg TDS (4800 mg) per day and the dose recommended for phase 2 was 800 mg TDS (2400 mg) per day. The three most common adverse events seen in the study were anemia (n = 9, 81.8%), nausea (n = 7, 63.6%), and fatigue (n = 6, 55.5%). Interpretation The recommended phase 2 dose of mebendazole is 1600 mg TDS with temozolomide and temozolomide‐radiation combination while the dose of 800 mg TDS needs to be used with single‐agent CCNU.
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spelling doaj-art-d0d509fdcef449d0816d5a6561a55a022025-08-20T02:40:11ZengWileyCancer Medicine2045-76342020-07-019134676468510.1002/cam4.3094Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade gliomaVijay M. Patil0Arti Bhelekar1Nandini Menon2Atanu Bhattacharjee3Vijai Simha4Ram Abhinav5Anuja Abhyankar6Epari Sridhar7Abhishek Mahajan8Ameya D. Puranik9Nilendu Purandare10Amit Janu11Ankita Ahuja12Rahul Krishnatry13Tejpal Gupta14Rakesh Jalali15Department of Medical Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Medical Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Medical Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaSection of Biostatistics Centre for Cancer Epidemiology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Medical Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Medical Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Medical Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Pathology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Radiology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Nuclear Medicine Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Nuclear Medicine Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Radiology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Radiology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Radiation Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Radiation Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaDepartment of Radiation Oncology Tata Memorial CentreHomi Bhabha National Institute (HBNI) Mumbai IndiaAbstract Background Relapsed high‐grade glioma has dismal outcomes. Mebendazole has shown promising activity against glioma in in‐vitro and in‐vivo studies. Hence, we undertook a phase 1 study to repurpose mebendazole in the treatment of glioblastoma. Methods We conducted a phase 1 study (accelerated titrated design 4) of mebendazole in patients with recurrent glioblastoma (GBM). Patients eligible for re‐irradiation were enrolled in arm A1 (radiation with concurrent temozolomide 75 mg/m2 daily during the course of radiation+mebendazole) while patients who were ineligible were enrolled in either arm B1 (CCNU 110 mg/m2 day 1, every 6 weekly + mebendazole) or arm C1 (temozolomide 200 mg/m2 day 1‐5, every 4 weekly + mebendazole). The primary endpoint of phase 1 was to identify the MTD of mebendazole in each combination. Findings 11 patients were enrolled in the whole study. MTD of mebendazole was not reached in arm A1 and C1 and hence the recommended dose for phase 2 was 1600 mg TDS (4800 mg) per day. The MTD of mebendazole in combination with CCNU was 1600 mg TDS (4800 mg) per day and the dose recommended for phase 2 was 800 mg TDS (2400 mg) per day. The three most common adverse events seen in the study were anemia (n = 9, 81.8%), nausea (n = 7, 63.6%), and fatigue (n = 6, 55.5%). Interpretation The recommended phase 2 dose of mebendazole is 1600 mg TDS with temozolomide and temozolomide‐radiation combination while the dose of 800 mg TDS needs to be used with single‐agent CCNU.https://doi.org/10.1002/cam4.3094CheckpointGlioblastomaHigh‐grade GliomaMebendazoleRecurrenceRepurposing
spellingShingle Vijay M. Patil
Arti Bhelekar
Nandini Menon
Atanu Bhattacharjee
Vijai Simha
Ram Abhinav
Anuja Abhyankar
Epari Sridhar
Abhishek Mahajan
Ameya D. Puranik
Nilendu Purandare
Amit Janu
Ankita Ahuja
Rahul Krishnatry
Tejpal Gupta
Rakesh Jalali
Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma
Cancer Medicine
Checkpoint
Glioblastoma
High‐grade Glioma
Mebendazole
Recurrence
Repurposing
title Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma
title_full Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma
title_fullStr Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma
title_full_unstemmed Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma
title_short Reverse swing‐M, phase 1 study of repurposing mebendazole in recurrent high‐grade glioma
title_sort reverse swing m phase 1 study of repurposing mebendazole in recurrent high grade glioma
topic Checkpoint
Glioblastoma
High‐grade Glioma
Mebendazole
Recurrence
Repurposing
url https://doi.org/10.1002/cam4.3094
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