Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions

Drug toxicity prediction plays a crucial role in the drug research and development process, ensuring clinical drug safety. However, traditional methods are hampered by high cost, low throughput, and uncertainty of cross-species extrapolation, which has become a key bottleneck restricting the efficie...

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Main Authors: Ruiqiu Zhang, Hairuo Wen, Zhi Lin, Bo Li, Xiaobing Zhou
Format: Article
Language:English
Published: MDPI AG 2025-06-01
Series:Toxics
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Online Access:https://www.mdpi.com/2305-6304/13/7/525
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author Ruiqiu Zhang
Hairuo Wen
Zhi Lin
Bo Li
Xiaobing Zhou
author_facet Ruiqiu Zhang
Hairuo Wen
Zhi Lin
Bo Li
Xiaobing Zhou
author_sort Ruiqiu Zhang
collection DOAJ
description Drug toxicity prediction plays a crucial role in the drug research and development process, ensuring clinical drug safety. However, traditional methods are hampered by high cost, low throughput, and uncertainty of cross-species extrapolation, which has become a key bottleneck restricting the efficiency of new drug research and development. The breakthrough development of Artificial Intelligence (AI) technology, especially the application of deep learning and multimodal data fusion strategy, is reshaping the scientific paradigm of drug toxicology assessment. In this review, we focus on the application of AI in the field of drug toxicity prediction and systematically summarize the relevant literature and development status globally in the past years. The application of various toxicity databases in the prediction was elaborated in detail, and the research results and methods for the prediction of different toxicity endpoints were analyzed in depth, including acute toxicity, carcinogenicity, organ-specific toxicity, etc. Furthermore, this paper discusses the application progress of AI technologies (e.g., machine learning and deep learning model) in drug toxicity prediction, analyzes their advantages and challenges, and outlines the future development direction. It aims to provide a comprehensive and in-depth theoretical framework and actionable technical strategies for toxicity prediction in drug development.
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spelling doaj-art-957a8abfec634e6cb25ba16575a8d9192025-08-20T03:07:57ZengMDPI AGToxics2305-63042025-06-0113752510.3390/toxics13070525Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future DirectionsRuiqiu Zhang0Hairuo Wen1Zhi Lin2Bo Li3Xiaobing Zhou4National Institutes for Food and Drug Control, Chinese Academy of Medical-Sciences and Peking Union Medical College, Beijing 100730, ChinaNational Center for Safety Evaluation of Drugs, National Institutes for Food and Drug Control, Beijing 100076, ChinaNational Center for Safety Evaluation of Drugs, National Institutes for Food and Drug Control, Beijing 100076, ChinaNational Institutes for Food and Drug Control, Chinese Academy of Medical-Sciences and Peking Union Medical College, Beijing 100730, ChinaNational Center for Safety Evaluation of Drugs, National Institutes for Food and Drug Control, Beijing 100076, ChinaDrug toxicity prediction plays a crucial role in the drug research and development process, ensuring clinical drug safety. However, traditional methods are hampered by high cost, low throughput, and uncertainty of cross-species extrapolation, which has become a key bottleneck restricting the efficiency of new drug research and development. The breakthrough development of Artificial Intelligence (AI) technology, especially the application of deep learning and multimodal data fusion strategy, is reshaping the scientific paradigm of drug toxicology assessment. In this review, we focus on the application of AI in the field of drug toxicity prediction and systematically summarize the relevant literature and development status globally in the past years. The application of various toxicity databases in the prediction was elaborated in detail, and the research results and methods for the prediction of different toxicity endpoints were analyzed in depth, including acute toxicity, carcinogenicity, organ-specific toxicity, etc. Furthermore, this paper discusses the application progress of AI technologies (e.g., machine learning and deep learning model) in drug toxicity prediction, analyzes their advantages and challenges, and outlines the future development direction. It aims to provide a comprehensive and in-depth theoretical framework and actionable technical strategies for toxicity prediction in drug development.https://www.mdpi.com/2305-6304/13/7/525Artificial Intelligencemachine learningdeep learningtoxicity endpointstransfer learning
spellingShingle Ruiqiu Zhang
Hairuo Wen
Zhi Lin
Bo Li
Xiaobing Zhou
Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions
Toxics
Artificial Intelligence
machine learning
deep learning
toxicity endpoints
transfer learning
title Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions
title_full Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions
title_fullStr Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions
title_full_unstemmed Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions
title_short Artificial Intelligence-Driven Drug Toxicity Prediction: Advances, Challenges, and Future Directions
title_sort artificial intelligence driven drug toxicity prediction advances challenges and future directions
topic Artificial Intelligence
machine learning
deep learning
toxicity endpoints
transfer learning
url https://www.mdpi.com/2305-6304/13/7/525
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AT hairuowen artificialintelligencedrivendrugtoxicitypredictionadvanceschallengesandfuturedirections
AT zhilin artificialintelligencedrivendrugtoxicitypredictionadvanceschallengesandfuturedirections
AT boli artificialintelligencedrivendrugtoxicitypredictionadvanceschallengesandfuturedirections
AT xiaobingzhou artificialintelligencedrivendrugtoxicitypredictionadvanceschallengesandfuturedirections