journal article·2025-10-09Open access

Enhancing Tree-Based Machine Learning for Personalized Drug Assignment

Katyna Sada Del Real, Angel Rubio

Applied Sciences· Volume 15· Issue 19

Abstract

Personalized drug selection is crucial for treating complex diseases such as Acute Myeloid Leukemia, where maximizing therapeutic efficacy is essential. Although precision medicine aims to tailor treatments to individual molecular profiles, existing machine learning models often fall short in selecting the best drug from multiple candidates. We present SEATS (Systematic Efficacy Assignment with Treatment Seats), which adapts conventional models like Random Forest and XGBoost for multiclass drug assignment by allocating probabilistic “treatment seats” to drugs based on efficacy. This approach helps models learn clinically relevant distinctions. Additionally, we assess an interpretable Optimal Decision Tree (ODT) model designed specifically for drug assignment. Trained on the BeatAML2 cohort and validated on the GDSC AML cell line dataset, integrating SEATS with Random Forest and XGBoost improved prediction accuracy and consistency. The ODT model offered competitive performance with clear, interpretable decision paths and minimal feature requirements, facilitating clinical use. SEATS reorients standard models towards personalized drug selection. Combined with the ODT framework it provides effective, interpretable strategies for precision oncology, underscoring the potential of tailored machine learning solutions in supporting real-world treatment decisions.
Source: crossref · Quality: baseline

Research context

OpenAlex topics: Computational Drug Discovery Methods · Bioinformatics and Genomic Networks · Pharmacogenetics and Drug Metabolism

SciVX discovery tags are terminology-based. OpenAlex topics are source-provided classifications.

Affiliations

Departamento de Ingeniería Biomédica y Ciencias, TECNUN, Universidad de Navarra, 20018 San Sebastian, Spain

Instituto de Ciencia de Datos e Inteligencia Artificial (DATAI), Universidad de Navarra, 31080 Pamplona, Spain

Funding

Cancer Research UK · Fundación Científica Asociación Española Contra el Cáncer · Italian Association for Cancer Research · Basque Government · Spanish Government

References (35)

Show source-provided references
1. Global, regional, and national burden of acute myeloid leukemia, 1990–2021: A systematic analysis for the global burden of disease study 202110.1186/s40364-024-00649-y ↗
2. Cancer statistics, 2022
3. SnapShot: Acute Myeloid Leukemia10.1016/j.ccr.2012.10.017 ↗
4. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel10.1182/blood-2016-08-733196 ↗
5. The road to precision oncology10.1038/ng.3796 ↗
6. National Cancer Institute, Genomic Data Commons, and National Institutes of Health (2025, October 08). Acute Myeloid Leukemia—Cancer Stat Facts, (n.d.), Available online: https://seer.cancer.gov/statfacts/html/amyl.html.
7. Machine learning in clinical decision making10.1016/j.medj.2021.04.006 ↗
8. Pathway-guided deep neural network toward interpretable and predictive modeling of drug sensitivity10.1021/acs.jcim.0c00331 ↗
9. Preclinical-to-clinical Anti-cancer Drug Response Prediction and Biomarker Identification Using TINDL10.1016/j.gpb.2023.01.006 ↗
10. Toward Explainable Anticancer Compound Sensitivity Prediction via Multimodal Attention-Based Convolutional Encoders10.1021/acs.molpharmaceut.9b00520 ↗
11. Jiang, L., Jiang, C., Yu, X., Fu, R., Jin, S., and Liu, X. (2022). DeepTTA: A transformer-based model for predicting cancer drug response. Brief. Bioinform., 23.10.1093/bib/bbac100 ↗
12. MOLI: Multi-omics late integration with deep neural networks for drug response prediction10.1093/bioinformatics/btz318 ↗
13. Predicting Drug Response and Synergy Using a Deep Learning Model of Human10.1016/j.ccell.2020.09.014 ↗
14. Improving Anticancer Drug Selection and Prioritization via Neural Learning to Rank10.1021/acs.jcim.3c01060 ↗
15. Lao, C., Zheng, P., Chen, H., Liu, Q., An, F., and Li, Z. (2024). DeepAEG: A model for predicting cancer drug response based on data enhancement and edge-collaborative update strategies. BMC Bioinform., 25.10.1186/s12859-024-05723-8 ↗
16. Huang, X., Huang, K., Johnson, T., Radovich, M., Zhang, J., Ma, J., and Wang, Y. (2021). ParsVNN: Parsimony visible neural networks for uncovering cancer-specific and drug-sensitive genes and pathways. NAR Genom. Bioinform., 3.10.1093/nargab/lqab097 ↗
17. Taj, F., and Stein, L.D. (2024). MMDRP: Drug response prediction and biomarker discovery using multi-modal deep learning. Bioinform. Adv., 4.10.1093/bioadv/vbae010 ↗
18. Kernelized rank learning for personalized drug recommendation10.1093/bioinformatics/bty132 ↗
19. Lenhof, K., Eckhart, L., Gerstner, N., Kehl, T., and Lenhof, H.P. (2022). Simultaneous regression and classification for drug sensitivity prediction using an advanced random forest method. Sci. Rep., 12.10.1038/s41598-022-17609-x ↗
20. Moughari, F.A., and Eslahchi, C. (2021). A computational method for drug sensitivity prediction of cancer cell lines based on various molecular information. PLoS ONE, 16.10.1371/journal.pone.0250620 ↗
21. Emdadi, A., and Eslahchi, C. (2020). DSPLMF: A Method for Cancer Drug Sensitivity Prediction Using a Novel Regularization Approach in Logistic Matrix Factorization. Front. Genet., 11.10.3389/fgene.2020.00075 ↗
22. Choi, J., Park, S., and Ahn, J. (2020). RefDNN: A reference drug based neural network for more accurate prediction of anticancer drug resistance. Sci. Rep., 10.10.1038/s41598-020-58821-x ↗
23. Zhang, F., Wang, M., Xi, J., Yang, J., and Li, A. (2018). A novel heterogeneous network-based method for drug response prediction in cancer cell lines. Sci. Rep., 8.10.1038/s41598-018-21622-4 ↗
24. Deep reinforcement learning for personalized treatment recommendation10.1002/sim.9491 ↗
25. Eckardt, J.-N., Wendt, K., Bornhäuser, M., Middeke, J.M., Stadlbauer, A., Meyer-Baese, A., and Zimmermann, M. (2021). Reinforcement Learning for Precision Oncology. Cancers, 13.10.3390/cancers13184624 ↗
26. Machine Learning for Clinical Outcome Prediction10.1109/RBME.2020.3007816 ↗
27. Machine learning in clinical practice: Prospects and pitfalls10.5694/mja2.50294 ↗
28. Integrative analysis of drug response and clinical outcome in acute myeloid leukemia10.1016/j.ccell.2022.07.002 ↗
29. Genomics of Drug Sensitivity in Cancer (GDSC): A resource for therapeutic biomarker discovery in cancer cells10.1093/nar/gks1111 ↗
30. Gimeno, M., José-Enériz, E.S., Fernandez, S.V., Agirre, X., Prosper, F., Rubio, A., and Carazo, F. (2022). Explainable Artificial Intelligence for Precision Medicine in Acute Myeloid Leukemia. Front. Immunol., 13.10.3389/fimmu.2022.977358 ↗
31. Random forests for genomic data analysis10.1016/j.ygeno.2012.04.003 ↗
32. Branson, N., Cutillas, P.R., and Bessant, C. (2023). Comparison of multiple modalities for drug response prediction with learning curves using neural networks and XGBoost. Bioinform. Adv., 4.10.1093/bioadv/vbad190 ↗
33. Gimeno, M., del Real, K.S., and Rubio, A. (2023). Precision oncology: A review to assess interpretability in several explainable methods. Brief. Bioinform., 24.10.1093/bib/bbad200 ↗
34. From local explanations to global understanding with explainable AI for trees10.1038/s42256-019-0138-9 ↗
35. Lundberg, S.M., and Allen, P.G. (2017). Consistent feature attribution for tree ensembles. arXiv.