Addressing the Cold-Start Problem for Personalized Combination Drug Screening

Antoine de Mathelin, Christopher Tosh, Wesley Tansey
Proceedings of the 20th Machine Learning in Computational Biology meeting, PMLR 311:230-239, 2025.

Abstract

Personalizing combination therapies in oncology requires navigating an immense space of possible drug and dose combinations, a task that remains largely infeasible through exhaustive experimentation. Recent developments in patient-derived models have enabled high-throughput ex vivo screening, but the number of feasible experiments is limited. Further, a tight therapeutic window makes gathering molecular profiling information (e.g. RNA-seq) impractical as a means of guiding drug response prediction. This leads to a challenging cold-start problem: how do we select the most informative combinations to test early, when no prior information about the patient is available? We propose a strategy that leverages a pretrained deep learning model built on historical drug response data. The model provides both embeddings for drug combinations and dose-level importance scores, enabling a principled selection of initial experiments. We combine clustering of drug embeddings to ensure functional diversity with a dose-weighting mechanism that prioritizes doses based on their historical informativeness. Retrospective simulations on large-scale drug combination datasets show that our method substantially improves initial screening efficiency compared to baselines, offering a viable path for more effective early-phase decision-making in personalized combination drug screens.

Cite this Paper


BibTeX
@InProceedings{pmlr-v311-mathelin25a, title = {Addressing the Cold-Start Problem for Personalized Combination Drug Screening}, author = {de Mathelin, Antoine and Tosh, Christopher and Tansey, Wesley}, booktitle = {Proceedings of the 20th Machine Learning in Computational Biology meeting}, pages = {230--239}, year = {2025}, editor = {Knowles, David A and Koo, Peter K}, volume = {311}, series = {Proceedings of Machine Learning Research}, month = {10--11 Sep}, publisher = {PMLR}, pdf = {https://raw.githubusercontent.com/mlresearch/v311/main/assets/mathelin25a/mathelin25a.pdf}, url = {https://proceedings.mlr.press/v311/mathelin25a.html}, abstract = {Personalizing combination therapies in oncology requires navigating an immense space of possible drug and dose combinations, a task that remains largely infeasible through exhaustive experimentation. Recent developments in patient-derived models have enabled high-throughput ex vivo screening, but the number of feasible experiments is limited. Further, a tight therapeutic window makes gathering molecular profiling information (e.g. RNA-seq) impractical as a means of guiding drug response prediction. This leads to a challenging cold-start problem: how do we select the most informative combinations to test early, when no prior information about the patient is available? We propose a strategy that leverages a pretrained deep learning model built on historical drug response data. The model provides both embeddings for drug combinations and dose-level importance scores, enabling a principled selection of initial experiments. We combine clustering of drug embeddings to ensure functional diversity with a dose-weighting mechanism that prioritizes doses based on their historical informativeness. Retrospective simulations on large-scale drug combination datasets show that our method substantially improves initial screening efficiency compared to baselines, offering a viable path for more effective early-phase decision-making in personalized combination drug screens.} }
Endnote
%0 Conference Paper %T Addressing the Cold-Start Problem for Personalized Combination Drug Screening %A Antoine de Mathelin %A Christopher Tosh %A Wesley Tansey %B Proceedings of the 20th Machine Learning in Computational Biology meeting %C Proceedings of Machine Learning Research %D 2025 %E David A Knowles %E Peter K Koo %F pmlr-v311-mathelin25a %I PMLR %P 230--239 %U https://proceedings.mlr.press/v311/mathelin25a.html %V 311 %X Personalizing combination therapies in oncology requires navigating an immense space of possible drug and dose combinations, a task that remains largely infeasible through exhaustive experimentation. Recent developments in patient-derived models have enabled high-throughput ex vivo screening, but the number of feasible experiments is limited. Further, a tight therapeutic window makes gathering molecular profiling information (e.g. RNA-seq) impractical as a means of guiding drug response prediction. This leads to a challenging cold-start problem: how do we select the most informative combinations to test early, when no prior information about the patient is available? We propose a strategy that leverages a pretrained deep learning model built on historical drug response data. The model provides both embeddings for drug combinations and dose-level importance scores, enabling a principled selection of initial experiments. We combine clustering of drug embeddings to ensure functional diversity with a dose-weighting mechanism that prioritizes doses based on their historical informativeness. Retrospective simulations on large-scale drug combination datasets show that our method substantially improves initial screening efficiency compared to baselines, offering a viable path for more effective early-phase decision-making in personalized combination drug screens.
APA
de Mathelin, A., Tosh, C. & Tansey, W.. (2025). Addressing the Cold-Start Problem for Personalized Combination Drug Screening. Proceedings of the 20th Machine Learning in Computational Biology meeting, in Proceedings of Machine Learning Research 311:230-239 Available from https://proceedings.mlr.press/v311/mathelin25a.html.

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