Organizations: City University of Hong Kong, Hong Kong, PR China · City University of Hong Kong Shenzhen Research Institute, Shenzhen, PR China · The Hong Kong University of Science and Technology (Guangzhou), Guangzhou PR China · GreenDynamics, Sydney, NSW, Australia
Recent advances in large language models have demonstrated considerable potential in scientific domains such as drug repositioning. However, their effectiveness remains constrained when reasoning extends beyond the knowledge acquired during pretraining. Conventional approaches, such as fine-tuning or retrieval-augmented generation, face limitations in either imposing high computational overhead or failing to fully exploit structured scientific data. To overcome these challenges, we propose DrugMCTS, a novel framework that synergistically integrates RAG, multi-agent collaboration, and Monte Carlo Tree Search for drug repositioning. The framework employs five specialized agents tasked with retrieving and analyzing molecular and protein information, thereby enabling structured and iterative reasoning. Extensive experiments on the DrugBank and KIBA datasets demonstrate that DrugMCTS achieves substantially higher recall and robustness compared to both general-purpose LLMs and deep learning baselines. Our results highlight the importance of structured reasoning, agent-based collaboration, and feedback-driven search mechanisms in advancing LLM applications for drug repositioning.
Figures & tables
Figure 1: When the document relevant to the query appears in the middle of the retrieved document sequence, the model’s performance can even fall below that of the baseline closed-book setting [ 16 ] .
Figure 2: Data processing pipiline of DrugMCTS
Figure 3: Illustration of a single rollout in the DrugMCTS framework. The figure demonstrates how agents sequentially collaborate during one inference trajectory guided by MCTS.
Figure 4: Example of MCTS for drug repositioning
Setup
S1 Baseline
S2 EI
S3 MAE
S4 IAE
S5 DE
S6 RR
DrugMCTS
DrugBank
0.1285
0.1586
0.3879/0.4677
0.3946/0.5119
0.3472/0.3617
0.4320/0.5527
0.4466/0.5534
KIBA
0.2284
0.2452
0.3772/0.4352
0.3846/0.4491
0.3189/0.3264
0.4193/0.4861
0.4224/0.4924
Table 2: Performance comparison on DrugBank and KIBA datasets using Qwen7b (Top-k/Top-k+3 accuracy).
Appendix figures & tables7 assets
Supplementary material from the paper’s appendix.
Appendix
Dataset
Processed Points
All Proteins
Ground Truth
Unique Molecules
All Molecules
DrugBank
788
22508
1595
1304
7717
KIBA
626
23849
1664
752
6219
Appendix
Table 3: Experimental Dataset Statistics
Dataset
Ground Truth
All Proteins
Ratio (%)
DB
1595
14654
10.88
KIBA
1664
10593
15.71
Appendix
Table 4: Baseline Dataset Statistics
Dataset
Split
Negative
Positive
Total
DrugBank
Train
14,787
12,428
27,215
Test
1,960
4,111
6,071
KIBA
Train
62,553
17,350
79,903
Test
31,643
4,804
36,447
Appendix
Table 5: Deep Learning Dataset Statistics
Figure 5: Number of rollouts vs Recall score on (a) DrugBank and (b) KIBA dataset
Figure 6: Number of tokens vs Recall score on (a) DrugBank and (b) KIBA dataset
Figure 7: Protein-ligand docking results between (a) global binding site overview and (b) detailed interaction view, computed by AutoDock Vina with default scoring function and visualized using PyMOL.
Drug repurposing aims to identify new therapeutic uses for existing compounds and, compared with de novo drug discovery, offers a faster and more cost-effective path to clinical translation. However, the space of candidate drug-disease pairs is enormous and their underlying relationships often depend on complex multi-hop biological mechanisms, making it difficult to reliably predict which pairs represent true therapeutic relationships. Existing approaches tackle this from two directions: knowledge graph-based methods organize curated biomedical evidence into structured relational networks for grounded multi-hop reasoning, while LLM-based methods leverage pretrained knowledge to generate flexible mechanistic rationales. Yet neither is sufficient alone - KGs are confined to observed graph structure while LLMs lack factual grounding and risk hallucination. To address this gap, we propose DrugReason, a multi-view reasoning framework that integrates grounded KG reasoning with LLM-generated mechanistic inference for drug repurposing. DrugReason adaptively routes diverse reasoning paths to specialized experts conditioned on the query context, while a cross-expert distillation objective enables knowledge sharing without sacrificing expert specialization. Experiments on PharmaDB, DDInter, and DrugBank show that DrugReason improves average performance over strong single-view reasoning baselines and achieves competitive or superior results compared with graph-based alternatives, while providing interpretable routing-based predictions.
Zijie Liu, Hongxuan Li, Zhen Tan +5
University of North Carolina at Chapel Hill · Stevens Institute of Technology · Emory University +1
Large language models (LLMs) have recently shown strong progress on scientific reasoning, yet two major bottlenecks remain. First, explicit retrieval fragments reasoning, imposing a hidden "tool tax" of extra tokens and steps. Second, multi-agent pipelines often dilute strong solutions by averaging across all candidates. We address these challenges with a unified framework that combines implicit retrieval and structured collaboration. At its foundation, a Monitor-based retrieval module operates at the token level, integrating external knowledge with minimal disruption to reasoning. On top of this substrate, Hierarchical Solution Refinement (HSR) iteratively designates each candidate as an anchor to be repaired by its peers, while Quality-Aware Iterative Reasoning (QAIR) adapts refinement to solution quality. On Humanity's Last Exam (HLE) Bio/Chem Gold, our framework achieves 48.3% accuracy - the highest reported to date, surpassing the strongest agent baseline by 13.4 points and leading frontier LLMs by up to 18.1 points, while simultaneously reducing token usage by 53.5% and agent steps by 43.7%. Results on SuperGPQA and TRQA confirm robustness across domains. Error analysis shows that reasoning failures and knowledge gaps co-occur in over 85% of cases, while diversity analysis reveals a clear dichotomy: retrieval tasks benefit from solution variety, whereas reasoning tasks favor consensus. Together, these findings demonstrate how implicit augmentation and structured refinement overcome the inefficiencies of explicit tool use and uniform aggregation. The code is available at https://github.com/tangxiangru/Eigen-1.
Xiangru Tang, Wanghan Xu, Yujie Wang +13
Yale University · Shanghai Jiao Tong University · Fudan University +4
Tool-augmented large language model (LLM) agents promise to unify scientific reasoning with computation, yet their deployment in high-stakes domains like drug discovery is bottlenecked by two critical barriers: unconstrained tool-use governance and poor long-horizon reliability. In dependency-heavy pharmaceutical pipelines, autonomous agents often drift into irreproducible trajectories, where early-stage hallucinations multiplicatively compound into downstream failures. To overcome this, we present Mozi, a dual-layer architecture that bridges the flexibility of generative AI with the deterministic rigor of computational biology. Layer A (Control Plane) establishes a governed supervisor--worker hierarchy that enforces role-based tool isolation, limits execution to constrained action spaces, and drives reflection-based replanning. Layer B (Workflow Plane) operationalizes canonical drug discovery stages -- from Target Identification to Lead Optimization -- as stateful, composable skill graphs. This layer integrates strict data contracts and strategic human-in-the-loop (HITL) checkpoints to safeguard scientific validity at high-uncertainty decision boundaries. Operating on the design principle of ``free-form reasoning for safe tasks, structured execution for long-horizon pipelines,'' Mozi provides built-in robustness mechanisms and trace-level audibility to completely mitigate error accumulation. We evaluate Mozi on PharmaBench, a curated benchmark for biomedical agents, demonstrating superior orchestration accuracy over existing baselines. Furthermore, through end-to-end therapeutic case studies, we demonstrate Mozi's ability to navigate massive chemical spaces, enforce stringent toxicity filters, and generate highly competitive in silico candidates, effectively transforming the LLM from a fragile conversationalist into a reliable, governed co-scientist.