By capitalizing on the wealth of existing pharmacological knowledge and embracing new scientific methodologies, drug repurposing holds tremendous promise for accelerating the development of effective, accessible, and affordable treatments for a wide range of diseases.
Drug Repurposing
Researched by:
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Karen Pendergrass
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Karen Pendergrass
Karen Pendergrass is a microbiome researcher specializing in microbiome-targeted interventions (MBTIs). She systematically analyzes scientific literature to identify microbial patterns, develop hypotheses, and validate interventions. As the founder of the Microbiome Signatures Database, she bridges microbiome research with clinical practice. In 2012, based on her own investigative research, she became the first documented case of FMT for Celiac Disease—four years before the first published case study.
Drug repurposing involves identifying new therapeutic uses for existing drugs, offering a cost-effective and time-efficient pathway to enhance treatment options and address unmet medical needs.
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Karen Pendergrass
Researched by:
-
Karen Pendergrass
ID
Karen Pendergrass
Microbiome Signatures identifies and validates condition-specific microbiome shifts and interventions to accelerate clinical translation. Our multidisciplinary team supports clinicians, researchers, and innovators in turning microbiome science into actionable medicine.
Karen Pendergrass
Overview Overview
Drug repurposing, or reprofiling, is a strategic approach in pharmaceutical and medical research that addresses the high costs and lengthy timelines typical of traditional drug discovery by leveraging existing drugs for new therapeutic uses. This method offers considerable advantages, such as significantly reduced cost and time to market, since these drugs have well-documented safety profiles and pharmacokinetics, allowing them to bypass early-stage trials and focus on proving efficacy for new indications. This is especially crucial in areas like oncology, where the need for new therapies is urgent, but the approval rate for novel drugs is low.
What are the major benefits of drug repurposing?
| Key Points | Benefits of Drug Repurposing |
|---|---|
| Reduces Risks | Drug repurposing reduces the risks associated with new drug development, particularly those arising from safety and efficacy issues. |
| Increases Success Likelihood | Starting with already approved drugs increases the likelihood of successful outcomes for new applications. |
| Innovative and Efficient Strategies | The shift towards drug repurposing reflects a move in the pharmaceutical industry towards innovative and efficient drug development strategies. |
| Cost-effective Solutions | Drug repurposing provides cost-effective solutions that accelerate the availability of therapies and enhance patient outcomes. |
| Efficiency and Cost-effectiveness | This approach significantly reduces the time and financial resources required to bring a treatment to market by leveraging existing data on drug safety and efficacy. |
| Enhanced Drug Discovery Process | Advanced data mining techniques and computational methods, such as artificial intelligence, modernize the identification of new therapeutic uses for existing drugs. |
| Broadening Treatment Horizons | Drug repurposing is particularly beneficial in areas with limited treatment options, addressing needs in rare diseases, complex conditions, or novel pathogens. |
| Scientific and Clinical Innovation | Exploring off-target effects and fostering interdisciplinary collaboration in repurposing efforts drives scientific and clinical innovation. |
| Regulatory and Developmental Advantages | Repurposed drugs often face a more straightforward pathway to approval for new uses due to prior extensive testing and regulatory scrutiny, facilitating faster access to critical medications. |
While drug repurposing offers significant advantages, it also poses substantial challenges, particularly in relation to intellectual property rights. The limited patent protection afforded to repurposed drugs can inhibit investment, underscoring the necessity for policies and incentives that bolster innovation in this field.
What are the other names for drug repurposing?
In the realm of pharmaceuticals, terms like drug repurposing, repositioning, reprofiling, and re-tasking are used interchangeably, each highlighting a different aspect of the strategy to apply existing drugs to new medical uses. Despite their subtle differences, these terms share a common goal: to innovate within pharmacological research by efficiently and cost-effectively extending the therapeutic uses of known drugs, blending disciplines like clinical medicine, pharmacology, computational biology, and bioinformatics.
| TermDefinition and ContextDrug RepurposingBroadly describes the process of finding new therapeutic uses for existing drugs or compounds beyond their original medical indications. It encompasses various strategies employed to identify and validate new uses for existing drugs.Drug RepositioningOften synonymous with drug repurposing, repositioning emphasizes the strategic aspect of changing a drug’s therapeutic use based on insights into its pharmacodynamics, pharmacokinetics, and off-target effects.Drug ReprofilingInvolves a detailed re-evaluation of a drug’s profile, including its mechanism of action, potential targets, and side effects, aimed at identifying new therapeutic applications. This term implies a depth of analysis using computational biology, systems biology, and other high-throughput techniques.Drug Re-taskingLess commonly used, re-tasking refers to assigning a new task or purpose to an existing drug. It highlights the practical aspect of using a known drug to address a different health condition, akin to assigning a new role to an existing resource. |
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Future
The future of drug repurposing is poised at an exciting juncture, leveraging advanced technologies and methodologies to streamline the discovery and development of new therapeutic uses for existing drugs. This paradigm shift from traditional discovery processes, which are often lengthy and costly, to more efficient and cost-effective strategies is catalyzed by significant advancements in data analytics, computational biology, and systems pharmacology.
What key trends are shaping innovations in drug repurposing?
The trajectory of drug repurposing is shaped by several key trends and innovations:
| Methodology | Description |
|---|---|
| Integration of Big Data and Computational Methods | Leveraging large-scale biomedical datasets and sophisticated computational algorithms, including machine learning, network analysis, and data mining, to discover precise drug-disease associations and uncover hidden relationships between drugs and new therapeutic targets. |
| High Throughput Screening (HTS) | HTS technologies facilitate the rapid evaluation of thousands of compounds against specific biological targets or disease models, efficiently identifying promising repurposing candidates and reducing development time and costs. |
| Systematic Drug Repurposing | Utilizes a holistic approach to analyze the complex networks of genes, proteins, and metabolic pathways, helping to pinpoint effective repurposing opportunities and visualize drug-disease connections at a systemic level through tools like network analysis. |
| Collaborative and Open Science Initiatives | Benefits from partnerships among academic institutions, pharmaceutical companies, and government agencies. Open science initiatives enhance innovation and speed up the discovery of repurposed drugs by promoting data and resource sharing. |
| Regulatory Support and Incentives | Regulatory bodies like the FDA provide guidance and incentives, such as fast-track designations and orphan drug status, to encourage drug repurposing efforts, especially for addressing unmet medical needs and expediting the approval process. |
| Patient-driven Initiatives | Emphasizes patient advocacy and patient-centric approaches, prioritizing repurposing opportunities that address direct patient needs, particularly for rare or neglected diseases, and mobilizing resources for potentially transformative treatments. |
FDA-Approved Discoveries
Numerous drugs have been successfully repurposed and are now FDA-approved for new indications beyond their original uses. For instance, Galantamine, initially marketed for paralysis, is now used for central nervous system conditions, while Dimethyl fumarate, first used for psoriasis, is approved for multiple sclerosis. Other notable examples include Cyclosporine, originally for transplant rejection now used for psoriasis, and Sildenafil, initially for angina now widely known for treating erectile dysfunction. These examples highlight the significant potential of drug repurposing in extending therapeutic options across various diseases.
What are some examples of drug repurposing that have been FDA-Approved?
| Condition | Drug | First Marketed For |
|---|---|---|
Central nervous system (CNS) | Galantamine | Paralysis |
| Dimethyl fumarate | Psoriasis | |
| Propranalol | Hypertension supra- ventricular tachycardia prolonged Q-T interval thyrotoxicosis | |
| Amantidine | Antiviral | |
| Respiratory System | Glycopyrronium | Peptic Ulcers |
| Psoriasis | Cyclosporine | Rheumatoid arthritis |
| Methotrexate | Cancer | |
| Plaque psoriasis | Etanercept | Rheumatoid arthritis |
| Rheumatoid arthritis | Rituximab | Cancer |
| Methotrexate | Cancer | |
| Neonatal-onset multi-system inflammatory disease | Anakinra | Rheumatoid arthritis |
| Gastrointestinal stromal tumor | Imatinib | Chronic myelogousleukaemia |
| Influenza A | Amantadine | Parkinson’s disease |
| Antiplatelet | Organ transplant rejection | Inflammation, pain relief |
| Transplant rejection | Cyclosporine | Rheumatoid arthritis |
| Alopecia | Minoxidil | Hypertension |
| Leprosy | Clofazime | Tuberculosis |
| Fibromyalgia | Milnacipran | Depression |
| Duloxetine | Depression | |
| Pregabalin | Anti-convulsant and neuropathic pain | |
| Atopic dermatitis | Doxepin | Depression |
| HIV/AIDS | Zidovudine | Cancer |
| Type II diabetes | Bromocriptine | Parkinson’s disease |
| Diabetic neuropathic pain | Duloxetine | Depression |
| Multiple myeloma | Thalidomide | Morning sickness |
| Hirutism | Eflornithine | Cancer |
| Pemphigus vulgaris | Rituximab | Cancer |
| Menopausal hot flashes | Paroxetine | Anti-depressant |
| Erectile dysfunction | Sildenafil | Angina |
| Erytheme nodosum leprosum | Thalidomide | Morning sickness |
| Autoimmune lympho-proliferative syndrome (ALPS) | Rapamycin | Organ transplantrejection |
| Multiple Sclerosis | Dimethylfumarate | Psoriasis |
| Leishmaniasis | Amphotericin | Anti-fungal |
| Miltefosine | Cancer | |
| Human African trypanosomiasis | Eflornithine | Cancer |
Erectile Dysfunction
Erectile dysfunction (ED) involves the consistent inability to sustain an erection, frequently connected to broader health issues and disturbances in the gut microbiome.
Erectile Dysfunction
Erectile dysfunction (ED) involves the consistent inability to sustain an erection, frequently connected to broader health issues and disturbances in the gut microbiome.