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Updated: Apr 4, 2026

Using Human Differentially Expressed Gene Lists to Perform Downstream Pathway Enrichment Analysis and Target Prioritization
Published on: October 3, 2025
A method for predicting target drug efficiency in cancer based on the analysis of signaling pathway activation
Artem Artemov1,2, Alexander Aliper2,3, Michael Korzinkin1
1Pathway Pharmaceuticals, Wan Chai, Hong Kong, Hong Kong SAR.
Abstract:
A new generation of anticancer therapeutics called target drugs has quickly developed in the 21st century. These drugs are tailored to inhibit cancer cell growth, proliferation, and viability by specific interactions with one or a few target proteins. However, despite formally known molecular targets for every "target" drug, patient response to treatment remains largely individual and unpredictable. Choosing the most effective personalized treatment remains a major challenge in oncology and is still largely trial and error. Here we present a novel approach for predicting target drug efficacy based on the gene expression signature of the individual tumor sample(s). The enclosed bioinformatic algorithm detects activation of intracellular regulatory pathways in the tumor in comparison to the corresponding normal tissues. According to the nature of the molecular targets of a drug, it predicts whether the drug can prevent cancer growth and survival in each individual case by blocking the abnormally activated tumor-promoting pathways or by reinforcing internal tumor suppressor cascades. To validate the method, we compared the distribution of predicted drug efficacy scores for five drugs (Sorafenib, Bevacizumab, Cetuximab, Sorafenib, Imatinib, Sunitinib) and seven cancer types (Clear Cell Renal Cell Carcinoma, Colon cancer, Lung adenocarcinoma, non-Hodgkin Lymphoma, Thyroid cancer and Sarcoma) with the available clinical trials data for the respective cancer types and drugs. The percent of responders to a drug treatment correlated significantly (Pearson's correlation 0.77 p = 0.023) with the percent of tumors showing high drug scores calculated with the current algorithm.
Insights
This study introduces a novel bioinformatic algorithm to predict targeted cancer drug efficacy using gene expression signatures. The approach accurately forecasts patient response, improving personalized oncology treatment strategies.
Area of Science:
- Oncology
- Bioinformatics
- Genomics
Background:
- Targeted cancer drugs offer personalized treatment by inhibiting specific molecular targets.
- However, predicting individual patient response to these therapies remains a significant clinical challenge.
- Current treatment selection often relies on trial and error.
Purpose of the Study:
- To develop and validate a novel bioinformatic algorithm for predicting targeted drug efficacy.
- To personalize cancer treatment by analyzing individual tumor gene expression signatures.
- To correlate predicted drug efficacy with clinical trial outcomes.
Main Methods:
- A bioinformatic algorithm was developed to detect aberrant intracellular regulatory pathway activation in tumor samples compared to normal tissues.
- The algorithm predicts drug efficacy by assessing the potential of a drug to block tumor-promoting pathways or activate tumor suppressor cascades.
- Predicted efficacy scores for five targeted drugs across seven cancer types were compared with clinical trial data.
Main Results:
- The algorithm successfully identified pathway activation patterns indicative of drug response.
- A significant positive correlation (Pearson's r = 0.77, p = 0.023) was observed between predicted high drug efficacy scores and the percentage of clinical trial responders.
- This validates the algorithm's ability to predict patient response to targeted anticancer therapies.
Conclusions:
- The developed gene expression-based algorithm provides a promising tool for predicting targeted drug efficacy in individual cancer patients.
- This approach has the potential to significantly improve personalized treatment selection in oncology.
- Accurate prediction of treatment response can reduce trial-and-error approaches and optimize patient outcomes.
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13:34A Combined 3D Tissue Engineered In Vitro/In Silico Lung Tumor Model for Predicting Drug Effectiveness in Specific Mutational Backgrounds
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