Network Dysregulation Analysis for De Novo Drug MoA Detection

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Solution Overview

Problem

Current methods for identifying the mechanism of action (MoA) of compounds are challenging, expensive, and often miss indirect or low-affinity targets, and are limited by their reliance on in vitro studies and prior knowledge, making them unsuitable for genome-wide analyses or de novo MoA elucidation.

Innovation Solution

A method called DeMAND, which uses network dysregulation analysis by processing gene expression data from tissue-specific regulatory networks to identify MoA by determining statistically significant dysregulated interactions and ranking candidate genes, leveraging Kullback-Leibler divergence and Brown's method for statistical significance.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Measurement precision

If direct binding assays are used to identify high-affinity binding targets, then binding target identification is improved, but indirect effectors and low-affinity binding targets are missed

Engineering Contradiction:
Improvebinding target identificationVSAvoidindirect effectors and low-affinity binding targets
Core Design Contradiction:
Measurement precisionVSLoss of information

Solution Approach 1:

The method segments the MoA identification process into two complementary approaches: direct binding assays for high-affinity targets and gene expression profiling for indirect effectors and low-affinity targets. This segmentation allows each method to focus on its strength while the results are integrated to provide comprehensive coverage of all MoA components.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The invention merges direct binding assay data with gene expression profiling data to create a unified MoA identification framework. By combining these two data sources, the method recovers both direct binding targets and indirect effectors that would be missed by either approach alone, thereby reducing information loss.

Inventive Principle:
Principle #5Merging (Combining)

2Reliability

If in vitro studies are used for MoA analysis, then controlled experimental conditions are achieved, but complex in vivo effects are missed

Engineering Contradiction:
Improveexperimental controlVSAvoidparacrine, endocrine, and contact signals in vivo
Core Design Contradiction:
ReliabilityVSLoss of information

Solution Approach 1:

The method adds a new dimension to MoA analysis by integrating gene expression data that reflects in vivo physiological responses. This dimensional expansion allows the detection of systemic effects including paracrine, endocrine, and contact signals that occur in living organisms but cannot be captured in isolated in vitro systems.

Inventive Principle:
Principle #17Another dimension (Dimensionality change)

3Measurement precision

If chemo-informatics methods rely on detailed three-dimensional structures and prior knowledge, then structural and genomic information integration is improved, but de novo MoA elucidation is limited

Engineering Contradiction:
Improvestructural and genomic information integrationVSAvoidde novo MoA elucidation
Core Design Contradiction:
Measurement precisionVSAdaptability or versatility

Solution Approach 1:

The method performs preliminary action by building comprehensive gene expression profiles and regulatory networks beforehand. These pre-established resources enable rapid de novo MoA elucidation for new compounds without requiring prior structural knowledge or extensive three-dimensional modeling, thereby enhancing adaptability while maintaining precision.

Inventive Principle:
Principle #10Preliminary action

4Reliability

If network-based methods require very large samples sizes, then statistical robustness is improved, but practical application to small compound libraries is made difficult

Engineering Contradiction:
Improvestatistical robustnessVSAvoidanalysis efficiency for small compound libraries
Core Design Contradiction:
ReliabilityVSProductivity

Solution Approach 1:

The method applies partial action by using a reduced but sufficient sample size tailored to the specific application. Rather than requiring very large sample sizes for all cases, the approach uses the minimum necessary samples to achieve adequate statistical robustness, thereby improving productivity for small compound libraries while maintaining reliability where needed.

Inventive Principle:
Principle #16Partial or excessive action

Data Source

PatentEP3140648B1Methods and systems for identifying a drug mechanism of action using network dysregulation
Publication Date: 2025.07.16 THE TRUSTEES OF COLUMBIA UNIV IN THE CITY OF NEW YORK
  • EP3140648B1 patent drawingFigure 1
  • EP3140648B1 patent drawingFigure 2
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AI summary

Techniques to identify a mechanism of action of a compound using network dysregulation are disclosed herein. An example method can include selecting at least a first interaction involving at least a first gene, determining a first n-dimensional probability density of gene expression levels for the first gene and one or more genes in a control state, determining a second n-dimensional probability density of gene expression levels for the first gene and one or more genes following treatment using at least one compound, estimating changes between the first probability density and the second probability density, and determining whether the estimated changes are statistically significant.