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Updated: Nov 5, 2025

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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
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Drug Repurposing: A Network-based Approach to Amyotrophic Lateral Sclerosis
Giulia Fiscon1,2, Federica Conte1, Susanna Amadio3
1Institute for Systems Analysis and Computer Science "A. Ruberti", National Research Council (IASI-CNR), Via Dei Taurini 19, 00185, Rome, Italy.
Summary
This study repurposed drugs for amyotrophic lateral sclerosis (ALS) using network medicine. Modafinil and other non-customary drugs showed promise for treating this progressive neurological disease.
Area of Science:
- Neuroscience
- Pharmacology
- Computational Biology
Background:
- Amyotrophic lateral sclerosis (ALS) is a progressive, debilitating neurological disease with no cure.
- The conventional "one target, one drug" approach has been ineffective for complex diseases like ALS.
Purpose of the Study:
- To identify novel therapeutic strategies for ALS by employing Network Medicine and drug repurposing.
- To discover new drug-ALS disease associations beyond traditional treatment paradigms.
Main Methods:
- Utilized SAveRUNNER, a network-based algorithm, to quantify gene-drug target proximity in the human interactome.
- Prioritized 403 drugs based on network similarity to ALS.
- Performed gene set enrichment analysis to validate predictions.
Main Results:
- Identified several non-customary drugs, including amoxapine, clomipramine, mianserin, and modafinil, as potential ALS therapeutics.
- Modafinil emerged as a top-scoring drug among 121 identified candidates.
- Network analysis revealed potential drug-ALS associations across various receptor modulators and enzyme inhibitors.
Conclusions:
- Network Medicine and drug repurposing offer innovative therapeutic avenues for ALS.
- Modafinil and other identified drugs warrant further investigation for ALS treatment.
- This study provides evidence for novel therapeutic solutions in ALS pathogenesis.

