DRUG REPURPOSING: OpportunitIes for new indication... ‘A SERENDIPITOUS PROCESS’ ABSTRACT: Introduction Drug repurposing is the technique of using an existing drug or drug candidate for a new treatment or medical condition for which it was not indicated before. This process can be called as a second life for drugs. It has been described as a serendipitous process that happens unexpectedly. In this process, the undesired side effects of drug molecules can also be a pointer to exploring the possibility of its effectiveness in an entirely different medical condition. This method emerges as a promising solution to this dilemma of traditional drug discovery, which has been a long laborious and costly process, often taking years or even decades to bring a new drug to the market. The principles of drug repurposing revolve around utilizing existing drug knowledge, employing advanced data analysis tools, identifying potential therapeutic targets, and gaining mechanistic insights. By harnessing the power of existing drugs and comprehensive data analyses, drug repurposing offers a strategic and efficient approach to discovering new therapeutic applications for the benefit of patients and healthcare systems. OBJECTIVES success stories 1. Reduce Time and Cost of Drug Development 2. Improve Success Rates 3. Address Unmet Medical Needs 4. Optimize Drug Utilization and Efficacy 5. Leverage Known Mechanisms of Action 6. Enhance Drug Safety and Reduce Adverse Effects 7. Increase Return on Investment for Pharmaceutical Companies 8. Respond Rapidly to Emerging Health Threats 9. Facilitate Personalized Medicine 10. Encourage Scientific Innovation and Collaboration 1.Metformin primarily used to treat Type II Diabetes is repurposed to treat Polycystic Ovary Syndrome (PCOS). 2. Thalidomide used for treating nausea in pregnancy(was banned) is repurposed for Leprosy and Multiple Myeloma. 3. Viagra (Sildenafil) used in Angina and Hypertension is repurposed to treat Erectile dysfunction. 4.Dexamethasone is corticosteroid(Rheumatoid arthritis, Asthama) that was repurposed for the treatment of severe COVID-19 cases. approaches 1. Computational Approaches: In silico screening, data mining, machine learning, network pharmacology. 2. Experimental Approaches: High-throughput screening (HTS) and phenotypic screening. 3. Clinical Approaches: Retrospective clinical analysis, off-label use analysis, observational studies. 4. Drug Combination Approaches: Drug combination screening for complex diseases like cancer or neurodegenerative disorders. 5. Pathway and Mechanism-Based Approaches: Pathway-based repurposing and off-target activity exploration. challenges 1. Lack of Pre-clinical studies. 2. Drug identification accuracy. 3. Intellectual Property Rights (IPR) and Patent issues. 4. Financial risks. 5. Market demand uncertainty. 6. Regulatory hurdles requiring new clinical trials. why drug repurposing? Repurposing can identify new compounds based on phenotypic benefits without explicitly defining the mechanism of action. This can be directly tested in pre-clinical animal models and these results are more applicable to clinical applications and research. It may progress directly to Phase II clinical trials. There is a minimum risk of failure with repurposed drugs. Here's a graph which explains that drug repurposing is significantly faster, cheaper and has a higher success rate as compared to developing a new drug from scratch. future opportunities STAGES: 1. Enhanced Computational Methods 2. Comprehensive Databases 3. Incentives for rare diseases 4. Focus on Combination Therapies 5. Precision Medicine Integration 6. Patient-Centric Trials 7. Intellectual Property Reforms Reference: 1. Kulkarni VS, Alagarsamy V, Solomon VR, Jose PA, Murugesan S. Drug repurposing: an effective tool in modern drug discovery. Russian Journal of Bioorganic Chemistry. 2023 Apr;49(2):157-66. 2. Shoaib M, Amjad Kamal M, Mohd Danish Rizvi S. Repurposed drugs as potential therapeutic candidates for the management of Alzheimer's disease. Current Drug Metabolism. 2017 Sep 1;18(9):842-52. Conclusion There are numerous diseases for which good therapeutic options have not been developed. The concept of repurposing a drug enables exploring the hidden potential of many molecules and better utilization of therapeutic agents. For better drug repositioning, more in-depth understanding along with integrated approaches between computational and experimental methods may be required to ensure high success rates of repositioned drugs. Name: KETAKI S. PATIL Year of study: Iv year College: ADITYA PHARMACY COLLEGE