Drug combination-wide association studies of cancer

Abstract Background Combinations of common drugs may, when taken together, have unexpected effects on incidence of diseases like cancer. It is not feasible to test for all combination drug effects in clinical trials, but in the real world, drugs are frequently taken in combination. Then, undiscovere...

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Main Authors: Panagiotis Nikolaos Lalagkas, Rachel Dania Melamed
Format: Article
Language:English
Published: Nature Portfolio 2025-07-01
Series:Communications Medicine
Online Access:https://doi.org/10.1038/s43856-025-00991-8
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author Panagiotis Nikolaos Lalagkas
Rachel Dania Melamed
author_facet Panagiotis Nikolaos Lalagkas
Rachel Dania Melamed
author_sort Panagiotis Nikolaos Lalagkas
collection DOAJ
description Abstract Background Combinations of common drugs may, when taken together, have unexpected effects on incidence of diseases like cancer. It is not feasible to test for all combination drug effects in clinical trials, but in the real world, drugs are frequently taken in combination. Then, undiscovered effects may protect users of drug combinations from cancer—or increase their risk. By analyzing massive health data containing numerous people exposed to drug combinations, we have an opportunity to discover these associations. Method We describe, apply, and evaluate an approach for discovering drug combination associations with cancer using health data. Our approach builds on marginal structural model methods to emulate a randomized trial where one arm is assigned to take a drug alone, while the other arm takes that drug in combination with a second drug. Results Here, we perform drug combination-wide analysis to estimate effects of over 9000 drug combinations on incidence of all common cancer types, using claims data covering more than 100 million people. But, because the discovery of associations from observational data is always prone to confounding, we develop a number of strategies to distinguish confounding from biomedically relevant findings. We describe a robustly supported beneficial drug combination that may synergistically impact lipid levels to reduce the risk of cancer. Conclusions These findings can suggest new clinical uses for drug combinations to prevent or treat cancer. Our approach can be adapted to mine electronic health records for interactive effects on other late-onset common diseases.
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spelling doaj-art-c9552a58117145bdacbfd77dc81632f52025-08-20T03:46:13ZengNature PortfolioCommunications Medicine2730-664X2025-07-015111210.1038/s43856-025-00991-8Drug combination-wide association studies of cancerPanagiotis Nikolaos Lalagkas0Rachel Dania Melamed1Department of Biological Sciences, University of MassachusettsDepartment of Biological Sciences, University of MassachusettsAbstract Background Combinations of common drugs may, when taken together, have unexpected effects on incidence of diseases like cancer. It is not feasible to test for all combination drug effects in clinical trials, but in the real world, drugs are frequently taken in combination. Then, undiscovered effects may protect users of drug combinations from cancer—or increase their risk. By analyzing massive health data containing numerous people exposed to drug combinations, we have an opportunity to discover these associations. Method We describe, apply, and evaluate an approach for discovering drug combination associations with cancer using health data. Our approach builds on marginal structural model methods to emulate a randomized trial where one arm is assigned to take a drug alone, while the other arm takes that drug in combination with a second drug. Results Here, we perform drug combination-wide analysis to estimate effects of over 9000 drug combinations on incidence of all common cancer types, using claims data covering more than 100 million people. But, because the discovery of associations from observational data is always prone to confounding, we develop a number of strategies to distinguish confounding from biomedically relevant findings. We describe a robustly supported beneficial drug combination that may synergistically impact lipid levels to reduce the risk of cancer. Conclusions These findings can suggest new clinical uses for drug combinations to prevent or treat cancer. Our approach can be adapted to mine electronic health records for interactive effects on other late-onset common diseases.https://doi.org/10.1038/s43856-025-00991-8
spellingShingle Panagiotis Nikolaos Lalagkas
Rachel Dania Melamed
Drug combination-wide association studies of cancer
Communications Medicine
title Drug combination-wide association studies of cancer
title_full Drug combination-wide association studies of cancer
title_fullStr Drug combination-wide association studies of cancer
title_full_unstemmed Drug combination-wide association studies of cancer
title_short Drug combination-wide association studies of cancer
title_sort drug combination wide association studies of cancer
url https://doi.org/10.1038/s43856-025-00991-8
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