Editorial - Journal of Experimental Stroke & Translational Medicine (2025) Volume 17, Issue 1
Drug Repurposing: Accelerating Innovation in Therapeutics
Laura Chen*
Department of Pharmacology, National University of Singapore, Singapore
- *Corresponding Author:
- Laura Chen
Department of Pharmacology, National University of Singapore, Singapore
E-mail: laura.chen@nus.edu.sg
Received: 01-Jan-2025, Manuscript No. jestm-25-170375; Editor assigned: 3-Jan-2025, PreQC No. jestm-25-170375 (PQ); Reviewed: 17-Jan-2025, QC No. jestm-25-170375; Revised: 22-Jan-2025, Manuscript No. jestm-25-170375 (R); Published: 29-Jan-2025, DOI: 10.37532/jestm.2024.16(6).307-308
Abstract
Introduction
Drug discovery is a long, costly, and high-risk process, often taking more than a decade and billions of dollars to bring a new therapy from concept to market. Despite these investments, many drug candidates fail during clinical development due to safety concerns or lack of efficacy [1]. To address these challenges, drug repurposing—also known as drug repositioning—has emerged as a promising strategy.
Drug repurposing involves finding new therapeutic uses for existing drugs, whether approved, investigational, or shelved. Since these compounds already have established safety profiles, their development for new indications can be faster, less expensive, and more efficient than starting from scratch. Over the past two decades, this approach has yielded several notable successes and is now viewed as a cornerstone of modern pharmacological innovation.
Rationale for Drug Repurposing
Traditional drug discovery focuses on identifying new chemical entities for specific molecular targets. However, biological systems are interconnected, and drugs often interact with multiple pathways beyond their original targets. This property, known as polypharmacology, creates opportunities to reposition existing drugs for alternative conditions.
The advantages of drug repurposing include:
Reduced Risk: Established pharmacokinetic and safety data minimize unexpected adverse effects.
Cost-Effectiveness: Shorter development timelines lower research and development expenses [2].
Faster Access for Patients: Particularly valuable for rare diseases and urgent health crises.
Novel Insights: Repurposing can uncover unexpected disease mechanisms and expand therapeutic options.
Methods of Drug Repurposing
Computational Approaches: Bioinformatics and artificial intelligence allow researchers to analyze large-scale datasets, such as genomics, proteomics, and clinical records, to identify potential drug-disease associations. Network pharmacology and machine learning are increasingly powerful tools in this space.
Experimental Approaches: High-throughput screening of existing drug libraries against disease models can reveal new therapeutic activities. Advances in patient-derived organoids and animal models support this method.
Clinical Observation: Serendipitous discoveries often arise from clinical practice, where unexpected drug effects suggest new uses [3]. For example, sildenafil was initially investigated for hypertension but later repurposed for erectile dysfunction and pulmonary hypertension.
Text Mining and Data Reuse: Mining biomedical literature and analyzing electronic health records can reveal off-label uses or correlations that inform repurposing candidates.
Success Stories in Drug Repurposing
Thalidomide: Originally introduced as a sedative and withdrawn due to teratogenic effects, thalidomide was later repurposed for multiple myeloma and leprosy-related complications.
Metformin: Initially developed for diabetes management, metformin is now being explored for cancer prevention, aging, and cardiovascular disease.
Minoxidil: First tested as an antihypertensive, it was repurposed for hair growth and remains a widely used treatment for alopecia.
Remdesivir: Initially developed for Ebola, it was rapidly repurposed during the COVID-19 pandemic as an antiviral option [4].
These examples highlight the diverse therapeutic opportunities that repurposing can unlock.
Challenges and Limitations
Despite its promise, drug repurposing is not without barriers:
Intellectual Property Issues: Patents for older drugs may have expired, limiting commercial incentives for pharmaceutical companies.
Regulatory Pathways: Although safety profiles exist, repurposed drugs still require rigorous testing and approval for new indications.
Biological Complexity: Not all off-target interactions are beneficial; some may cause harmful side effects.
Data Limitations: Many computational predictions require extensive validation before clinical translation.
Future Directions
The future of drug repurposing lies in integrating advanced technologies with collaborative frameworks:
Artificial Intelligence and Multi-Omics: Combining big data analytics with genomics, proteomics, and metabolomics will refine candidate identification [5].
Global Databases and Sharing Initiatives: Open-access drug libraries and clinical trial repositories will accelerate discovery.
Precision Medicine: Repurposing efforts tailored to genetic and molecular patient profiles can maximize therapeutic success.
Public-Private Partnerships: Collaboration between academia, industry, and government agencies will be crucial to overcoming regulatory and financial barriers.
Conclusion
Drug repurposing exemplifies how innovation can arise from reimagining existing resources. By harnessing established compounds for new therapeutic applications, this approach reduces development time, lowers costs, and expands treatment options for patients. While intellectual property, regulatory, and biological challenges remain, advances in computational biology, systems pharmacology, and collaborative research continue to push the field forward. In an era where efficiency and precision are critical, drug repurposing stands as a vital strategy for accelerating global healthcare innovation.
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