Predicting Drug Response and Synergy Using a Deep Learning Model of Human Cancer Cells

Brent M Kuenzi1, Jisoo Park1, Samson H Fong2

  • 1Division of Genetics, Department of Medicine, University of California San Diego, La Jolla, CA 92093, USA.

Cancer Cell
|October 23, 2020
PubMed

Insights

DrugCell, an interpretable deep learning model, accurately predicts cancer drug responses and identifies synergistic drug combinations by analyzing tumor genotypes and drug structures. This approach enhances predictive medicine and aids in clinical trial success.

Area of Science:

  • Computational Biology
  • Pharmacology
  • Machine Learning

Background:

  • High failure rates in drug clinical trials stem from poor understanding of drug response mechanisms.
  • Current machine learning models lack interpretability and focus on monotherapies, limiting clinical application.

Purpose of the Study:

  • To develop DrugCell, an interpretable deep learning model for predicting human cancer cell drug response.
  • To elucidate the biological mechanisms underlying drug response and identify synergistic drug combinations.

Main Methods:

  • Trained DrugCell on 1,235 tumor cell lines and 684 drugs, integrating tumor genotypes with drug structures.
  • Utilized deep learning for predictive modeling and mechanism discovery.
  • Validated synergistic drug combinations using combinatorial CRISPR, in vitro screening, and patient-derived xenografts.

Main Results:

  • DrugCell demonstrated accurate drug response predictions in cell lines.
  • Model predictions successfully stratified clinical outcomes.
  • Identified and validated synergistic drug combinations with significant therapeutic potential.

Conclusions:

  • DrugCell offers an interpretable framework for predictive medicine in oncology.
  • The model facilitates the discovery of novel therapeutic strategies, including synergistic drug combinations.
  • This approach provides a blueprint for developing interpretable AI models in clinical practice.