Introduction to Pharmacology and Drug Discovery
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Questions and Answers

What is the primary focus of pharmacodynamics?

  • The adverse effects of drugs
  • How drugs affect physiological systems (correct)
  • How drugs are used in clinical practice
  • The study of drugs from natural sources

    • Which of the following is an advantage of protein-based therapeutics?

  • They are more effective for all diseases
  • They have no side effects
  • They have a longer shelf life than chemical drugs
  • They can be specifically designed to target certain cells (correct)

    • What characterizes target-led drug discovery?

  • It aims to understand the biological mechanisms involved in diseases (correct)
  • It involves screening large libraries of compounds
  • It is based on the identification of disease-causing agents
  • It primarily focuses on the chemical structure of compounds

    • What are monoclonal antibodies primarily used for?

    <p>To target and destroy specific cells or pathogens</p> Signup and view all the answers

    Which area of therapeutics is NOT mentioned as overlapping with pharmacology?

    <p>Psychological therapy</p> Signup and view all the answers

    What was the primary focus of Sir James Black's program established in 1964?

    <p>Synthesis of H2 receptor antagonists</p> Signup and view all the answers

    What was the significance of proposing a new histamine receptor (H2) in 1948?

    <p>It offered a solution for treating peptic ulcers.</p> Signup and view all the answers

    Which compound showed a significant difference in H2 selectivity compared to histamine?

    <p>Burimamide</p> Signup and view all the answers

    What was noted as a potential issue with Metiamide?

    <p>Granulocytopenia</p> Signup and view all the answers

    In the context of the study, what does a high value of the dissociation constant indicate?

    <p>Low efficacy as an antagonist</p> Signup and view all the answers

    What is an example of a drug derived from a plant?

    <p>Digitalis</p> Signup and view all the answers

    Which statement accurately describes drugs compared to herbal medicine?

    <p>Drugs have a known dose.</p> Signup and view all the answers

    Which of the following is a characteristic of homeopathy?

    <p>Unregulated manufacture and sale</p> Signup and view all the answers

    In drug discovery, which approach involves identifying a drug target of interest?

    <p>Target-led discovery</p> Signup and view all the answers

    What type of activity do traditional drugs exhibit?

    <p>Biological effect</p> Signup and view all the answers

    Which of the following is true about the regulation of drugs?

    <p>Drugs are generally well-regulated.</p> Signup and view all the answers

    What is a characteristic of drugs when compared to homeopathy?

    <p>Drugs have biological activity.</p> Signup and view all the answers

    Which of the following statements is true about the characteristics of microbe-derived drugs?

    <p>Microbe-derived drugs can have a single active component.</p> Signup and view all the answers

    What is a characteristic of stem cells?

    <p>They can transform into various tissue-specific cell types.</p> Signup and view all the answers

    What does CAR-T cell therapy involve?

    <p>Genetically modifying patients' T cells to target cancer cells.</p> Signup and view all the answers

    What is a primary application of cell-based therapies?

    <p>Introduction of genetically-modified cells to treat disease.</p> Signup and view all the answers

    How have the regulations for medical devices and drugs changed recently?

    <p>The division between the two areas has blurred.</p> Signup and view all the answers

    What is a challenge associated with traditional stents in cardiology?

    <p>They can lead to vessel re-occlusion.</p> Signup and view all the answers

    What is a major disadvantage of using purified insulin from blood donations?

    <p>Risk of blood donation infections</p> Signup and view all the answers

    What is the primary benefit of recombinant proteins over naturally purified proteins?

    <p>Limited potential for viral contamination</p> Signup and view all the answers

    What key breakthrough did Köhler and Milstein achieve in 1975?

    <p>Immortalization of antibody-producing cells</p> Signup and view all the answers

    What is an advantage of monoclonal antibody therapies?

    <p>Predictable batch properties</p> Signup and view all the answers

    Which type of antibodies are denoted with the suffix -umab?

    <p>Human antibodies</p> Signup and view all the answers

    What is a disadvantage of using antivenin derived from horse serum?

    <p>Risk of immune reactions to foreign serum</p> Signup and view all the answers

    What role do antisense molecules play as drugs?

    <p>Bind to and inhibit transcription</p> Signup and view all the answers

    PATISARAN is an FDA-approved drug for which condition?

    <p>Hereditary amyloidosis with polyneuropathy</p> Signup and view all the answers

    What aspect does pharmacognosy primarily focus on within the study of pharmacology?

    <p>The study of drugs from naturally occurring sources</p> Signup and view all the answers

    Which of the following best describes the primary focus of pharmacotherapeutics?

    <p>How drugs are used in clinical settings</p> Signup and view all the answers

    Which statement reflects a common misconception about gene-based therapies?

    <p>They are always more effective than traditional therapies</p> Signup and view all the answers

    What is a notable disadvantage associated with the use of protein-based therapeutics?

    <p>They tend to have a shorter half-life than small molecule drugs</p> Signup and view all the answers

    What distinguishes target-led drug discovery from chemistry-led drug discovery?

    <p>Target-led relies heavily on understanding biological mechanisms</p> Signup and view all the answers

    Which of the following accurately describes a characteristic of stem cells?

    <p>They can differentiate into various mature tissue-specific cell types.</p> Signup and view all the answers

    What fundamental mechanism does CAR-T cell therapy employ to combat cancer?

    <p>It modifies the patient’s T cells to target specific tumor antigens.</p> Signup and view all the answers

    Which statement correctly identifies a difference between medical devices and drugs?

    <p>Devices are assessed based on performance, while drugs are evaluated for efficacy.</p> Signup and view all the answers

    In the context of cell-based therapies, which statement is true about the types of stem cells used?

    <p>Multipotent stem cells can differentiate into a limited range of cell types.</p> Signup and view all the answers

    What is one of the primary uses of adoptive cell transfer therapy?

    <p>To utilize genetically altered cells to enhance immune response against tumors.</p> Signup and view all the answers

    What is one major challenge in developing small molecule drugs?

    <p>Inability to target specific proteins</p> Signup and view all the answers

    Which therapeutic approach is NOT considered a current strategy in drug development?

    <p>Small molecule inhibitors</p> Signup and view all the answers

    What does the high value of the dissociation constant suggest about a compound?

    <p>Lower efficacy in therapeutic use</p> Signup and view all the answers

    What is a characteristic associated with proteins used as drugs?

    <p>They are always less effective than small molecules</p> Signup and view all the answers

    Which statement about the origins of modern drug development is correct?

    <p>The first drugs were isolated from natural sources</p> Signup and view all the answers

    In the context of drug interactions involving cytochrome P450, what does a lower dissociation constant imply?

    <p>Stronger competitive inhibition</p> Signup and view all the answers

    What distinct feature characterizes nucleic acid-based therapeutics?

    <p>They can provide targeted gene modulation</p> Signup and view all the answers

    What limitation do small molecule drugs face in terms of their mechanism of action?

    <p>They cannot bind to larger protein complexes</p> Signup and view all the answers

    What was the main therapeutic focus of the compounds developed in Sir James Black's program in 1964?

    <p>Antagonism of H2 receptors</p> Signup and view all the answers

    Which of the following compounds exhibited the highest selectivity for H2 receptors based on the presented dissociation constants?

    <p>Burimamide</p> Signup and view all the answers

    What adverse effect was noted in relation to the compound Metiamide?

    <p>Granulocytopenia</p> Signup and view all the answers

    In terms of oral absorption, which compound was noted for its poor absorption in the development of H2 antagonists?

    <p>Burimamide</p> Signup and view all the answers

    What was revealed about the dissociation constants of histamine and Burimamide concerning H1 and H2 receptors?

    <p>Burimamide has higher H2 selectivity compared to histamine.</p> Signup and view all the answers

    What is a potential risk associated with the use of traditional antivenin derived from horse serum?

    <p>Possible immune reactions to foreign serum</p> Signup and view all the answers

    Which recombinant protein is NOT typically produced through recombinant DNA technology?

    <p>Monoclonal antibodies</p> Signup and view all the answers

    What is a significant advantage of using recombinant proteins over purified hormones?

    <p>Lower dependency on human blood donations</p> Signup and view all the answers

    In monoclonal antibody nomenclature, which suffix indicates a chimeric antibody?

    <p>-ximab</p> Signup and view all the answers

    What is a disadvantage of monoclonal antibody therapies compared to traditional treatments?

    <p>Expensive production costs</p> Signup and view all the answers

    What function do 'antisense' molecules serve in therapeutic applications?

    <p>Inhibit transcription of target mRNA</p> Signup and view all the answers

    What is a limitation of using monoclonal antibodies in therapy?

    <p>They often require intravenous administration</p> Signup and view all the answers

    What characteristic is associated with recombinant protein therapies that reduces viral contamination risk?

    <p>Cells are used to generate recombinant proteins</p> Signup and view all the answers

    Study Notes

    Pharmacology Introduction

    • Pharmacology: the study of drugs and their effects on living organisms
    • Pharmacology sub-disciplines:
      • Pharmacokinetics: what happens to drugs in the body
      • Pharmacodynamics: what drugs do in the body
      • Pharmacotherapeutics: how drugs are used
      • Pharmacognosy: study of drugs from naturally occurring sources
      • Toxicology: study of adverse drug effects

    Drug Discovery Approaches

    • Chemistry-led Discovery:
      • Uses existing chemicals with biological activity
      • Modifies chemical structures to create a library of analogues
      • Screens analogues for biological activity
      • Generates structure-activity relationships (SARs)
      • Mechanism of action is often unknown
    • Target-led Discovery:
      • Identifies a drug target of interest
      • Screens chemicals for binding to the target
      • Designs chemical entities based on computer modelling
        • ligand docking to 3D protein structures
      • Mechanism is known, and effect can be predicted
      • Example: Receptor structure modelling

    Modern Drug Development

    • Protein-based therapeutics
      • Advantages:
        • Can treat complex diseases
        • Lower risk of triggering an immune response than traditional drugs
        • Relatively safe
      • Disadvantages:
        • Expensive to produce
        • Can be difficult to administer
        • Require specific storage conditions

    Monoclonal Antibodies

    • Antibodies: large proteins that recognise and bind to specific targets.
    • Monoclonal antibodies: Antibodies that all bind to the same epitope (target) on an antigen (protein).
    • Discovery: Köhler and Milstein (1975), using a method to immortalise antibody-producing cells (hybridoma technology)
    • Monoclonal antibodies are used for the treatment of a wide range of diseases such as cancer, autoimmune diseases, and infections.
    • Advantages:
      • High specificity and affinity for their target
      • Produces predictable and reliable results
      • Can be produced in large scale.
    • Disadvantages:
      • Can be expensive to produce
      • Can cause some unwanted side effects, such as allergic reactions
      • Short half-life (degradation)

    Types of Monoclonal Antibodies

    • Mouse Moabs (omab): made entirely from mouse, can trigger immune responses
    • Chimeric antibodies (ximab): made from human and mouse components, less likely to trigger immune responses
    • Humanised antibodies (zumab): made from human and mouse components, less likely to trigger immune responses
    • Human antibodies (umab): made from entirely human components, least likely to trigger immune responses

    Other Therapeutics

    • Antivenin/Antivenom:

      • Used to treat snake and spider bites.
      • Serum from an immunized horse containing antibodies against the venom.
      • Potentially dangerous side effects: allergic reactions and serum sickness.

    • Nucleic acids as drugs:

      • Antisense molecules: molecules that bind to and degrade specific mRNA strands.
        • Example: PATISIRAN (RNAi) therapy for hereditary amyloidosis with polyneuropathy.
      • Aptamers: synthetic molecules that bind to specific targets, preventing them from reaching their targets.

    • Gene therapy:

      • Introduces genes to correct defective genes or introduce genes to fight disease.
      • Limited by delivery mechanisms and potential off-target effects.

    • Cell-based therapies: - Involves the use of cells to treat diseases. - Examples: - Stem cell therapy: Stem cells have the ability to differentiate into various cell types. - Potential for treating a wide range of diseases, including leukemia and degenerative diseases. - Adoptive cell transfer therapy: Patients' own T cells are genetically modified to express synthetic receptors that recognize tumour antigens, and then administered back to the patient to fight cancer.

    Medical Devices

    • Medical Devices vs Drugs:
      • Medical devices have different regulatory requirements than drugs.
      • The distinction between the two has blurred with the development of drug-eluting stents for cardiovascular disease.

    Summary of Learning Outcomes.

    • Explain the differences between chemistry-led and target-led drug discovery
      • Chemistry-led: Starts with a compound and explores its activity. Target-led: Starts with a target and finds compounds to interact with it.
    • Describe the advantages and disadvantages of protein-based therapeutics.
      • Advantages: Treat complex diseases, safe, lower risk of immune responses. Disadvantages: Expensive, difficult administration, specific storage requirements.
    • Explain the generation, activity, and applications of monoclonal antibodies.
      • Generated: Using hybridoma technology. Activity: Highly specific to one epitope. Applications: Treatment of a wide range of diseases.
    • Discuss new areas of therapeutics that overlap with pharmacology, such as cell therapies and gene-based therapies.
      • Cell therapies: Involves the use of cells to treat diseases. Gene therapies: Uses genes to treat disease by introducing the correct version of a gene or by targeting specific genes.

    What is Pharmacology?

    • The study of drugs and their effects on the body.
    • It involves understanding how drugs interact with biological systems, their uses in treating disease, and their potential adverse effects.
    • Includes pharmacology subfields such as pharmacokinetics (what happens to drugs in the body), pharmacodynamics (what drugs do in the body), pharmacotherapeutics (how drugs are used), pharmacognosy (study of drugs from naturally occurring sources), and toxicology (study of adverse effects).

    Evolution of Pharmacology

    • Early examples of pharmacology include:
      • Egyptians using inhaled herbs for asthma (3400BC)
      • The use of Ma Huang (ephedrine) to treat asthma in China (1000BC)
    • Modern pharmacology dates back to the 19th century with the isolation and understanding of compounds from natural sources such as morphine, quinine, and cocaine.
    • The synthesis of aspirin in 1897 marked a crucial point in the development of synthesized medicines.

    Chemistry-Based Drug Development

    • Ranitidine is a prime example of chemistry-led drug development, led by Sir James Black at SKF:
      • Ranitidine was developed as an antagonist for the H2 histamine receptor.
      • Initial research determined the existence of an H2 receptor that caused gastric acid secretion.
      • Scientists synthesized histamine analogues and tested their selectivity for H2 receptors, resulting in the development of cimetidine and ranitidine.

    Protein-Based Therapies

    • Protein therapies are becoming increasingly important in medicine.
    • Advantages:
      • Proteins can be effective and plentiful.
      • They are relatively easy to isolate and purify.
    • Disadvantages:
      • Early protein therapies required blood donations, leading to risks of infection.
      • They were difficult to modify further.
    • Recombinant proteins solve these problems:
      • They are produced through cloning protein-encoding genes into cell lines.
      • This process offers lower risk of contamination and allows for greater control over production

    Monoclonal Antibody Therapies

    • Monoclonal antibodies (MoAbs) are antibodies produced by a single clone of B lymphocytes.
    • Köhler and Milstein revolutionized MoAb production by discovering a method to immortalize antibody-producing cells.
    • They fused antibody-producing cells from the spleen with myeloma cells, resulting in hybridoma cells that can continuously produce large quantities of a single type of antibody.
    • This discovery paved the way for the development of monoclonal antibodies for various therapeutic applications.

    Using Monoclonal Antibodies to Fight Cancer

    • Monoclonal antibodies can be used to target specific cancerous cells and trigger immune responses.
    • MoAbs can directly inhibit the growth of cancer cells or act as "carrier molecules" delivering cytotoxic drugs or toxins to tumor cells.
    • This targeted approach minimizes damage to healthy tissues.

    Value of Monoclonal Antibody Therapies

    • Advantages:
      • They can target specific epitopes on a specific antigen, maximizing efficacy and minimizing side effects.
      • Production no longer requires animal use.
      • They can be manufactured in large quantities, ensuring consistent quality.
    • Disadvantages:
      • Production can be expensive.
      • Antibodies are typically administered intravenously.

    Monoclonal Antibody Nomenclature

    • MoAb nomenclature is based on the origin of the antibody:
      • Mouse MoAbs - omab: example - tositumomab
      • Chimeric antibodies - ximab: example - infliximab
      • Humanized antibodies - zumab: example - natalizumab, trastuzumab
      • Human antibodies - umab: example - adalimumab

    Antivenin/Antivenom

    • Antivenin is a type of therapy used to treat snake and spider bites.
    • Antivenom typically uses serum from a horse immunized with snake venom.
    • The horse serum is then administered to the patient to neutralize the venom.
    • However, horse serum can trigger immune reactions since it's foreign to the human body, leading to potential complications.

    Nucleic Acids as Drugs

    • The use of nucleic acids as drugs is a new and rapidly growing area of medicine.
    • Two main types of nucleic acid-based therapies are:
      • Antisense molecules: These are short, single-stranded nucleic acids that bind to and inhibit specific mRNA molecules, blocking the production of a targeted protein. Patisaran is an FDA-approved RNAi therapy for a type of hereditary amyloidosis.
      • Aptamers: These are short, single-stranded nucleic acid molecules that bind to specific target molecules (proteins or other nucleic acids) with high affinity and specificity. They can be used for diagnostic purposes or to disrupt the function of a specific molecule in the body.

    Gene Therapy

    • Gene therapy is an innovative therapeutic strategy designed to correct genetic defects responsible for disease.
    • It involves introducing a therapeutic gene into a patient's cells to replace or alter a defective gene.
    • This holds enormous potential for treating diseases like cystic fibrosis, hemophilia, and certain cancers.

    Cell-Based Therapies

    • Cell-based therapy involves using living cells to treat disease.
    • The two main approaches are:
      • Genetically modified cells: Cells are modified to express a therapeutic gene, leading to a specific therapeutic effect.
    • Stem cell therapy: Stem cells, with their potential to differentiate into various cell types, offer promising treatment options for a wide range of diseases.

    Stem Cell Therapy

    • Stem cells are undifferentiated cells with the capacity to self-renew and develop into various cell types.
    • They can be sourced from various sources, including embryonic and adult tissues.
    • Stem cell transplantation is currently a primary treatment for leukemia, with promising applications in repairing damaged tissues or organs in the future.

    Adoptive Cell Transfer Therapy

    • Adoptive cell transfer therapy is a type of immunotherapy using a patient's own immune cells, specifically T cells, to fight cancer.
    • CAR-T (Chimeric Antigen Receptor- T cell) therapy involves isolating a patient's T cells, genetically modifying them to express synthetic receptors targeting tumor antigens, and then re-infusing these modified cells into the patient. This approach enhances the T cells' ability to recognize and destroy cancer cells.

    Medical Devices vs. Drugs

    • Medical devices and drugs are regulated differently, but the distinction is becoming blurred as new technologies emerge.
    • Drug-coated stents are a prime example of this blurring line, as they combine a medical device (stent) with a drug (anti-proliferative agent) to prevent vessel re-occlusion.

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    Introduction To Pharmacology RCSI FFP1-54 2024 PDF

    Description

    This quiz covers the fundamentals of pharmacology, including its sub-disciplines such as pharmacokinetics, pharmacodynamics, and pharmacotherapeutics. Additionally, it explores the approaches to drug discovery, focusing on chemistry-led and target-led methods. Test your knowledge of how drugs affect living organisms and the processes involved in discovering new medications.

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