Division of Pre-clinical Innovation, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH), Rockville, Maryland 20850, United States.
The Graduate School of Biomedical Sciences, Departments of Pharmacological Sciences, Icahn School of Medicine at Mount Sinai, New York, New York 10029, United States.
J Chem Inf Model. 2023 Feb 13;63(3):846-855. doi: 10.1021/acs.jcim.2c01516. Epub 2023 Jan 31.
Inappropriate use of prescription drugs is potentially more harmful in fetuses/neonates than in adults. Cytochrome P450 (CYP) 3A subfamily undergoes developmental changes in expression, such as a transition from CYP3A7 to CYP3A4 shortly after birth, which provides a potential way to distinguish medication effects on fetuses/neonates and adults. The purpose of this study was to build first-in-class predictive models for both inhibitors and substrates of CYP3A7/CYP3A4 using chemical structure analysis. Three metrics were used to evaluate model performance: area under the receiver operating characteristic curve (AUC-ROC), balanced accuracy (BA), and Matthews correlation coefficient (MCC). The performance varied for each CYP3A7/CYP3A4 inhibitor/substrate model depending on the data set type, model type, rebalancing method, and specific feature set. For the active inhibitor/substrate data set, the optimal models achieved AUC-ROC values ranging from 0.77 ± 0.01 to 0.84 ± 0.01. For the selective inhibitor/substrate data set, the optimal models achieved AUC-ROC values ranging from 0.72 ± 0.02 to 0.79 ± 0.04. The predictive power of the optimal models was validated by compounds with known potencies as CYP3A7/CYP3A4 inhibitors or substrates. In addition, we identified structural features significant for CYP3A7/CYP3A4 selective or common inhibitors and substrates. In summary, the top performing models can be further applied as a tool to rapidly evaluate the safety and efficacy of new drugs separately for fetuses/neonates and adults. The significant structural features could guide the design of new therapeutic drugs as well as aid in the optimization of existing medicine for fetuses/neonates.
不当使用处方药物对胎儿/新生儿的潜在危害大于成人。细胞色素 P450(CYP)3A 亚家族在表达上发生发育变化,例如出生后不久从 CYP3A7 向 CYP3A4 的转变,这为区分药物对胎儿/新生儿和成人的影响提供了一种潜在方法。本研究旨在使用化学结构分析为 CYP3A7/CYP3A4 的抑制剂和底物建立首创的预测模型。使用三个指标来评估模型性能:接收器操作特征曲线下的面积(AUC-ROC)、平衡准确性(BA)和马修斯相关系数(MCC)。每种 CYP3A7/CYP3A4 抑制剂/底物模型的性能因数据集类型、模型类型、再平衡方法和特定特征集而异。对于活性抑制剂/底物数据集,最佳模型的 AUC-ROC 值范围为 0.77 ± 0.01 至 0.84 ± 0.01。对于选择性抑制剂/底物数据集,最佳模型的 AUC-ROC 值范围为 0.72 ± 0.02 至 0.79 ± 0.04。最优模型的预测能力通过具有已知效力的化合物作为 CYP3A7/CYP3A4 抑制剂或底物进行了验证。此外,我们确定了对 CYP3A7/CYP3A4 选择性或共同抑制剂和底物有重要意义的结构特征。总之,表现最佳的模型可进一步作为工具,分别快速评估新药对胎儿/新生儿和成人的安全性和疗效。显著的结构特征可以指导新治疗药物的设计,并有助于优化胎儿/新生儿的现有药物。