BBB-Nuke: Transport-Aware Prediction of Blood-Brain Barrier Penetration in Small Molecules
Abasciano, N.; Hadipour, H.; Poddar, A.; Rudrum, J.; Sobodu, T.
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Predicting blood-brain barrier (BBB) penetration remains a central challenge in CNS drug discovery. Existing computational models rely on physicochemical descriptors and are blind to active transport biology - the efflux pumps and carrier proteins that dominate drug exclusion at the BBB in vivo. We present BBB-Nuke, a modular prediction pipeline that integrates physicochemical scoring with explicit efflux transporter substrate modeling. The system computes ten molecular descriptors, predicts ionization state via a graph convolutional network, scores CNS-MPO desirability, and estimates substrate probability for seven efflux transporters (P-gp/MDR1, BCRP/ABCG2, MRP1, MRP2, MRP4, MATE1, OAT3) using Random Forest classifiers trained on curated ChEMBL bioactivity data. A gradient-boosted classifier trained on 67 features - ten physicochemical, seven efflux transporter probabilities, and fifty fingerprint-derived principal components - achieves an area under the receiver operating characteristic curve (AUROC) of 0.933 {+/-} 0.006 under five-fold cross-validation on 9,262 labeled compounds, and 0.810 on a fully held-out benchmark of 470 clinically validated compounds. In head-to-head comparisons, BBB-Nuke outperforms CNS-MPO, LightBBB, ADMETlab 2.0, and BBB-Score on both cross-validation and external test sets. We apply the pipeline to screen over one billion commercially available compounds from the Enamine REAL library and PubChem, identifying enriched regions of BBB-penetrant chemical space and characterizing the structural features that distinguish permeable from excluded molecules. BBB-Nuke is freely available as a Python package, REST API, and Model Context Protocol server.
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