Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial

Background: Precision cancer medicine (PCM) is key to advancing cancer treatment beyond the standard of care. We performed an explorative clinical trial, MEGALiT, to investigate the feasibility, safety, and clinical benefit of genomics-based PCM in advanced cancer. Methods: MEGALiT recruited adult...

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Main Authors: Lars Ny, Henrik Fagman, Johan Botling, Loviisa Mantovaara, Peter Asplund, Hannah Karlsson, Jennie Aust, Edvard Abel, Mats Hellström, Joakim Crona, Peter Nygren
Format: Article
Language:English
Published: Medical Journals Sweden 2025-06-01
Series:Acta Oncologica
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Online Access:https://medicaljournalssweden.se/actaoncologica/article/view/43366
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author Lars Ny
Henrik Fagman
Johan Botling
Loviisa Mantovaara
Peter Asplund
Hannah Karlsson
Jennie Aust
Edvard Abel
Mats Hellström
Joakim Crona
Peter Nygren
author_facet Lars Ny
Henrik Fagman
Johan Botling
Loviisa Mantovaara
Peter Asplund
Hannah Karlsson
Jennie Aust
Edvard Abel
Mats Hellström
Joakim Crona
Peter Nygren
author_sort Lars Ny
collection DOAJ
description Background: Precision cancer medicine (PCM) is key to advancing cancer treatment beyond the standard of care. We performed an explorative clinical trial, MEGALiT, to investigate the feasibility, safety, and clinical benefit of genomics-based PCM in advanced cancer. Methods: MEGALiT recruited adult patients with advanced solid tumors refractory to standard treatment. Tumor DNA from newly acquired biopsies or ctDNA were analyzed for alterations targetable with the PD-L1 inhibitor atezolizumab, the MEK inhibitor cobimetinib, the mTOR inhibitor everolimus, or the PARP-inhibitor niraparib. Any other ‘in study’ treatment was left to the discretion of the physician. Results: Outcome data are reported for 153 patients. The median age was 65 years and the most common diagnoses were colorectal, prostate, and ovarian cancer. The median time from study inclusion to the Molecular Tumor Board was 35 days for tumor sampling by biopsy and 21 days by ctDNA. Of the 44 patients allocated to a study drug, 38 started treatment. The median follow-up was 1.9 years. Of the patients on a study drug and evaluable for tumor response, 6% (2/32) had partial remission, and 25% (8/32) had disease control at 16 weeks. Median overall survival for patients starting a study drug was longer, 7.4 months, compared to 2.7 months for the 61 untreated patients (HR 0.43; log-rank p < 0.0001), but shorter than for the 50 patients receiving treatment of physician’s choice, 11.8 months (HR 0.55; log-rank p = 0.012). No significant procedure- or drug-related severe adverse events were observed. Interpretation: Genomics-guided treatment selection in advanced cancer is feasible and safe. However, evidence of patient benefit warrants further investigation.
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spelling doaj-art-bdb7429f3c074d8f8c21334787ee64a72025-08-20T02:32:30ZengMedical Journals SwedenActa Oncologica1651-226X2025-06-016410.2340/1651-226X.2025.43366Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trialLars Ny0Henrik Fagman1Johan Botling2Loviisa Mantovaara3Peter Asplund4Hannah Karlsson5Jennie Aust6Edvard Abel7Mats Hellström8Joakim Crona9Peter Nygren10Institute of Clinical Sciences, Department of Oncology, University of Gothenburg, Gothenburg, Sweden, Department of Oncology, Sahlgrenska University Hospital, Gothenburg, SwedenDepartment of Clinical Pathology, Sahlgrenska University Hospital, Gothenburg, Sweden; Department of Laboratory Medicine, Institute of Biomedicine, Sahlgrenska Center for Cancer Research, University of Gothenburg, Gothenburg, SwedenDepartment of Clinical Pathology, Sahlgrenska University Hospital, Gothenburg, Sweden; Department of Laboratory Medicine, Institute of Biomedicine, Sahlgrenska Center for Cancer Research, University of Gothenburg, Gothenburg, Sweden; Department of Immunology, Genetics and Pathology, Uppsala University, Uppsala, SwedenDepartment of Immunology, Genetics and Pathology, Uppsala University, Uppsala, SwedenDepartment of Blood and Tumor Diseases, section of Oncology, University Hospital, Uppsala, SwedenDepartment of Blood and Tumor Diseases, section of Oncology, University Hospital, Uppsala, SwedenInstitute of Clinical Sciences, Department of Oncology, University of Gothenburg, Gothenburg, Sweden, Department of Oncology, Sahlgrenska University Hospital, Gothenburg, SwedenInstitute of Clinical Sciences, Department of Oncology, University of Gothenburg, Gothenburg, Sweden, Department of Oncology, Sahlgrenska University Hospital, Gothenburg, SwedenDepartment of Immunology, Genetics and Pathology, Uppsala University, Uppsala, SwedenDepartment of Medical Sciences, Uppsala University, Uppsala, SwedenDepartment of Immunology, Genetics and Pathology, Uppsala University, Uppsala, SwedenBackground: Precision cancer medicine (PCM) is key to advancing cancer treatment beyond the standard of care. We performed an explorative clinical trial, MEGALiT, to investigate the feasibility, safety, and clinical benefit of genomics-based PCM in advanced cancer. Methods: MEGALiT recruited adult patients with advanced solid tumors refractory to standard treatment. Tumor DNA from newly acquired biopsies or ctDNA were analyzed for alterations targetable with the PD-L1 inhibitor atezolizumab, the MEK inhibitor cobimetinib, the mTOR inhibitor everolimus, or the PARP-inhibitor niraparib. Any other ‘in study’ treatment was left to the discretion of the physician. Results: Outcome data are reported for 153 patients. The median age was 65 years and the most common diagnoses were colorectal, prostate, and ovarian cancer. The median time from study inclusion to the Molecular Tumor Board was 35 days for tumor sampling by biopsy and 21 days by ctDNA. Of the 44 patients allocated to a study drug, 38 started treatment. The median follow-up was 1.9 years. Of the patients on a study drug and evaluable for tumor response, 6% (2/32) had partial remission, and 25% (8/32) had disease control at 16 weeks. Median overall survival for patients starting a study drug was longer, 7.4 months, compared to 2.7 months for the 61 untreated patients (HR 0.43; log-rank p < 0.0001), but shorter than for the 50 patients receiving treatment of physician’s choice, 11.8 months (HR 0.55; log-rank p = 0.012). No significant procedure- or drug-related severe adverse events were observed. Interpretation: Genomics-guided treatment selection in advanced cancer is feasible and safe. However, evidence of patient benefit warrants further investigation. https://medicaljournalssweden.se/actaoncologica/article/view/43366precision cancer medicinegenomicsclinical trialtargeted drugPD-1/PD-L1inhibitors
spellingShingle Lars Ny
Henrik Fagman
Johan Botling
Loviisa Mantovaara
Peter Asplund
Hannah Karlsson
Jennie Aust
Edvard Abel
Mats Hellström
Joakim Crona
Peter Nygren
Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial
Acta Oncologica
precision cancer medicine
genomics
clinical trial
targeted drug
PD-1/PD-L1inhibitors
title Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial
title_full Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial
title_fullStr Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial
title_full_unstemmed Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial
title_short Feasibility and outcome of genomics-guided treatment selection in advanced cancer – the MEGALiT explorative clinical trial
title_sort feasibility and outcome of genomics guided treatment selection in advanced cancer the megalit explorative clinical trial
topic precision cancer medicine
genomics
clinical trial
targeted drug
PD-1/PD-L1inhibitors
url https://medicaljournalssweden.se/actaoncologica/article/view/43366
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