GMS 6551 Final Exam

What is the chemical name of a drug?

Describes its chemical structure and composition following specific rules of chemical nomenclature.

What is the process of absorption in pharmacokinetics?

The process by which a drug enters the bloodstream from its site of administration.

What are biologics?

A class of drugs derived from living systems. They are more complex than small molecules and are produced through biological processes using living cells or organisms. Examples include vaccines, antibodies, and gene therapies.

What is an agonist in pharmacology?

A drug that binds to a specific receptor and activates it, producing a biological response.

What is an antagonist?

A drug that binds to a specific receptor but does not activate it. Instead, it blocks the receptor, preventing the binding and action of other substances.

What is the difference between positive and negative controls in experimental design?

A positive control is an experimental condition that is known to produce a specific result, while a negative control is an experimental condition that is expected to produce no result. Both types of controls are used to validate experimental results.

What is Good Laboratory Practice (GLP)?

A set of guidelines for conducting pre-clinical studies that ensure the quality, integrity, and reliability of experimental data.

What is Good Manufacturing Practice (GMP)?

A set of guidelines for manufacturing and quality control of drugs, biologics, and medical devices that ensure their safety, efficacy, and consistency.

What is a power analysis in experimental design?

A statistical method used to determine the sample size needed to detect a significant effect in an experiment.

What is molecular docking?

A technique used in CADD to predict the preferred orientation and binding affinity of a small molecule (ligand) within the binding site of a target protein.

What is blinding in experimental design?

A technique used to prevent bias in experimental design by keeping the researchers or participants unaware of which group they are in (e.g. treatment group or control group).

What is the difference between accuracy and precision in diagnostic testing?

Accuracy refers to how close a measured value is to a standard or known value, while precision refers to the degree to which several measurements provide answers very close to each other.

Is achieving complete selectivity always possible in drug targeting?

Achieving complete selectivity is challenging, and even with highly selective drugs, some degree of off-target effects or side effects may still occur due to the complex and interconnected nature of biological systems. Preclinical and clinical testing is conducted to assess a drug's selectivity, safety, and efficacy.

What is a proprietary name?

Also known as the brand name or trade name, is the name under which a pharmaceutical company markets and sells a particular drug.

What is a non-proprietary name?

Also known as the generic name, is the official name assigned to a drug by regulatory authorities. It is a universally recognized name for the drug, irrespective of the manufacturer or brand.

What is computer-aided drug design (CADD)?

An approach that uses computational methods to assist in the discovery and design of new drugs.

What are animal models used for in drug discovery?

Animal models are used in in vivo assays to evaluate the effects of drug candidates on the whole organism. They provide insights into pharmacokinetics, toxicology, efficacy, and safety of the drug.

What are some examples of pre-clinical approaches?

Animal models, in vitro assays, and computational modeling.

What are small molecule drugs?

Are composed of low molecular weight compounds with a defined chemical structure. They are typically synthesized chemically and interact with specific targets in the body to produce a therapeutic effect.

What is assay development in the context of drug discovery?

Assay development involves designing and optimizing experimental methods to measure the activity, potency, or efficacy of a drug candidate.

Why is assay development important in drug discovery?

Assay development is crucial for establishing reliable and reproducible methods to measure drug activity and characterize its effects on biological systems, aiding in the evaluation of drug candidates during the discovery and development process.

What are the categories of assays used in drug discovery?

Assays can be categorized into in vitro assays (such as test tube assays and cell culture assays), in vivo assays (using animal models), and ex vivo assays (involving living tissue samples).

How are cell culture assays used in drug discovery?

Cell culture assays use cultured cells that mimic the in vivo environment. Drug candidates are added to the cell cultures, and their effects on cell viability, proliferation, signaling pathways, or gene expression are measured.

What are some examples of non-animal approaches that can be used to replace animal models in pre-clinical research?

Cell, biochemical, and/or in silico simulations.

What ethical considerations are associated with in vivo assays?

Conducting experiments on animals raises ethical concerns, and regulations and guidelines are in place to ensure the ethical treatment of animals in research.

What are drug targets?

Drug targets are specific molecules, including enzymes, receptors, proteins, DNA, and RNA, with which drugs interact in the body.

How can selectivity be achieved in cancer treatment?

Drugs designed for cancer treatment can selectively target mutated or overexpressed proteins found in cancer cells, disrupting their signaling pathways or inhibiting their function. This selectively targets cancer cells while sparing normal healthy cells, reducing side effects.

How do drugs interact with receptors?

Drugs interact with receptors, which are proteins located on cells, by binding to them and activating or inhibiting signaling pathways, leading to physiological responses.

What is the role of enzymes as drug targets?

Enzymes, which are proteins that catalyze chemical reactions, can be targeted by drugs to modulate their activity and regulate biochemical processes.

What measures are taken to design drugs with optimal selectivity profiles?

Extensive research, including structural and computational studies, is conducted to design drugs with optimal selectivity profiles, reducing the likelihood of unintended interactions and adverse effects.

What factors influence disease targeting by pharmaceutical companies?

Factors include prevalence, unmet medical need, market size, feasibility, and regulatory considerations.

What is the focus of pharmacokinetics?

Focuses on the study of how drugs are absorbed, distributed, metabolized, and eliminated by the body.

Why do governments provide incentives for orphan diseases?

Governments provide incentives for orphan diseases to encourage drug development for small patient populations.

Why is testing new compounds important in SAR?

Helps identify compounds with improved biological activity, selectivity, or other desirable properties.

What does pharmacodynamics study?

How drugs exert their effects on the body at the molecular, cellular, and physiological levels.

What does pharmacology study?

How drugs interact with the body and how the body responds to those drugs.

What does pharmacogenetics explore?

How genetic variations influence an individual's response to drugs.

What are some examples of diagnostic tools?

Imaging techniques (such as X-rays or MRI), blood tests, and genetic tests.

What is the importance of using in vitro and ex vivo assays alongside in vivo studies?

In vitro and ex vivo assays complement and validate the findings of in vivo studies, offering a more comprehensive understanding of the drug's effects.

What is an advantage of in vitro assays?

In vitro assays allow for high throughput screening of a large number of compounds, accelerating the drug discovery process.

What is a key aspect of in vitro assays?

In vitro assays are performed in controlled laboratory environments, allowing for precise control over experimental variables and the study of specific interactions between the drug candidate and its target molecule.

What administrative benefits do in vitro assays offer?

In vitro assays do not involve ethical considerations or regulatory requirements associated with animal research, streamlining the experimental process and reducing administrative burdens.

What is a disadvantage of in vitro assays?

In vitro assays lack the complexity to fully replicate the biological interactions and physiological responses seen in living organisms.

Why may in vitro assays have limited physiological relevance?

In vitro assays may not accurately reflect the behavior of a drug candidate in vivo, as they cannot capture factors such as tissue architecture, systemic influences, and pharmacokinetics.

How are in vitro assays cost-effective?

In vitro assays require fewer resources compared to in vivo or ex vivo assays, making them relatively inexpensive to perform.

What should be done to validate the findings from in vitro assays?

In vitro assays should be followed by in vivo or ex vivo studies to validate the findings and gain a comprehensive understanding of a drug candidate's effects in a living organism.

How do in vivo assays differ from in vitro assays in terms of complexity?

In vivo assays involve the interaction of drugs with complex physiological systems, introducing additional variables and complexities compared to in vitro assays.

What is an advantage of in vivo assays?

In vivo assays provide a more accurate representation of how a drug or treatment will behave in humans due to the similarities between animal and human physiology.

What is the disadvantage of in vivo assays?

In vivo studies are generally more expensive than in vitro assays due to the costs associated with animal housing, specialized equipment, and compliance with ethical and regulatory requirements.

What is the role of toxicology in pharmacology?

Investigates the adverse effects of drugs and other substances on living organisms and assesses their potential risks and safety profiles.

What are ex vivo assays?

Involve the use of living tissue samples obtained from animals or humans. Tissue slices or explants are treated with the drug candidate to assess its effects on specific tissues or organs.

What is the role of metabolism in pharmacokinetics?

Involves the enzymatic conversion of drugs into metabolites, impacting their efficacy, toxicity, and duration of action.

What is the next step after identifying a lead compound in drug discovery?

It undergoes further optimization through medicinal chemistry techniques to improve its potency, selectivity, pharmacokinetic properties, and safety profile, with the aim of developing a potential drug candidate.

What are the steps involved in the SAR process?

Modification of the lead compound, testing new compounds, choosing the best compound, and further functionalization.

How can natural products contribute to the identification of lead compounds?

Natural products obtained from plants, algae, bacteria, and fungi can be screened or isolated to identify potential lead compounds with desired pharmacological properties.

What are some sources of lead compounds in drug discovery?

Natural products, chemical banks, and rational drug design approaches.

Why do pharmaceutical companies focus on diseases with a large market?

Pharmaceutical companies target diseases with a large market for potential financial returns.

What are some of the phases of clinical trials?

Phase I (safety and dosage), Phase II (efficacy and side effects), and Phase III (large-scale efficacy and safety).

What is the role of ethics in translational research?

Plays a critical role in translational research by ensuring that research is conducted in a manner that is respectful of human subjects, protects their rights and welfare, and promotes the public good.

What are some of the key components of experimental design in pre-clinical research?

Power analysis, randomization, blinding of investigators, reagent/assay validation, positive/negative controls, and inclusion/exclusion of data.

Why are pharmacokinetic studies important in drug development?

Provide valuable information for optimizing drug dosage regimens, predicting drug-drug interactions, and ensuring safe and effective drug use.

How do SAR studies contribute to drug design?

Provide valuable insights into the relationship between the structure and activity of compounds, guiding the design of new compounds with optimized properties and therapeutic potential.

What are the three Rs of animal research?

Reduce, Refine, and Replace.

What is selectivity in drug targeting?

Refers to a drug's ability to specifically interact with its intended target while minimizing interactions with other molecules in the body.

What is the target of drug action?

Refers to the specific molecule or biological entity that the drug interacts with to produce its effects, such as a receptor or an enzyme.

What are some of the regulatory requirements that impact the movement of pre-clinical studies into human trials?

Safety and efficacy testing, ethical considerations, and compliance with Good Laboratory Practices (GLP) and Good Manufacturing Practices (GMP).

What are the limitations of molecular docking?

Simplified models and the potential omission of important details that may affect the accuracy of predictions.

What is the difference between small molecules and biologics?

Small molecules are low molecular weight compounds with a defined chemical structure, while biologics are more complex drugs derived from living systems. Small molecules are typically synthesized chemically, while biologics are produced through biological processes.

What is the difference between small molecules and biologics?

Small molecules are typically low molecular weight compounds that can be synthesized chemically, while biologics are typically large, complex molecules that are produced using living cells or organisms.

What are some examples of therapeutic modalities?

Small molecules, biologics, gene therapies, and cell therapies.

How can drugs achieve selectivity in targeting non-human proteins?

Some drugs selectively target proteins unique to pathogens or disease-causing organisms, allowing inhibition or killing of the pathogen without affecting human proteins, reducing side effects. This approach is often used in antimicrobial therapies.

What are the advantages of molecular docking and CADD?

Speed and cost-effectiveness compared to experimental techniques.

What does SAR stand for in drug discovery?

Structure-Activity Relationship.

What are the four phases of translational research?

T0 (basic research), T1 (pre-clinical research), T2 (clinical research), and T3 (implementation research).

Why is target identification important in drug discovery?

Target identification is crucial in drug discovery as it helps focus the design and optimization of drug candidates by understanding the disease mechanism and pathways involved in order to develop effective treatments.

Why is achieving selectivity challenging when targeting human proteins?

Targeting human proteins involved in normal physiological processes can be challenging due to the similarity between the target protein and related proteins in the body, increasing the risk of off-target effects and potential side effects.

What are test tube assays?

Test tube assays are performed in a controlled laboratory environment using isolated proteins or biochemical systems. They involve mixing the drug candidate with the target protein or its components in test tubes or microplates.

What are some of the advantages of using animal models in pre-clinical research?

The ability to control experimental conditions, the ability to study complex biological systems, and the ability to evaluate the safety and efficacy of new therapies or diagnostic tools in a living organism.

What are some of the advantages of using in vitro assays in pre-clinical research?

The ability to study specific cellular or molecular processes, the ability to screen large numbers of compounds or samples, and the ability to reduce the use of animal models.

What is the goal of pharmaceutical companies in disease targeting?

The goal is to develop effective treatments, improve patient outcomes, and address health challenges.

What is the goal of SAR in drug discovery?

The goal of SAR is to optimize the lead compound and design new compounds with improved potency, selectivity, and other desired properties.

What is pharmacokinetics?

The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.

What is the purpose of high-throughput screening in identifying lead compounds?

These techniques are used to screen large chemical libraries or banks for compounds that exhibit specific biological activities or interactions with a target of interest, aiding in the identification of lead compounds.

How is a drug eliminated from the body?

Through various routes, including urine, feces, exhaled air, sweat, and breast milk.

What is the purpose of rigor and reproducibility in research?

To ensure that experimental results are accurate, reliable, and can be replicated by other researchers.

What is the purpose of regulatory approval?

To ensure that new therapies or diagnostic tools are safe and effective for use in human subjects, and to provide guidelines for their use in clinical practice.

What is the purpose of animal models in pre-clinical research?

To evaluate the safety and efficacy of new therapies or diagnostic tools before testing them in human subjects.

What is the goal of T2 research?

To evaluate the safety and efficacy of new therapies or diagnostic tools in human subjects.

What is the purpose of clinical trials?

To evaluate the safety and efficacy of new therapies or diagnostic tools in human subjects.

What is the purpose of in vitro assays in pre-clinical research?

To evaluate the safety and efficacy of new therapies or diagnostic tools using cells or tissues in a laboratory setting.

How is molecular docking used in drug discovery?

To gain insights into the binding interactions between small molecules and target proteins, guiding the design of potential drug candidates.

What is the goal of T0 research?

To generate new knowledge about the underlying mechanisms of disease.

What is the goal of T1 research?

To identify and optimize therapeutic modalities and pre-clinical approaches for treating human conditions or diseases.

What is the goal of T3 research?

To implement new therapies or diagnostic tools into clinical practice and evaluate their effectiveness in real-world settings.

What is the purpose of computational modeling in pre-clinical research?

To simulate biological processes or drug interactions using mathematical or computer-based models.

What is translational research?

Translational research is the process of applying knowledge from basic science research to develop new therapies or diagnostic tools for human diseases. [PDF introduction]

What are some of the regulatory agencies involved in approving new therapies or diagnostic tools (name 3)?

US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA).

What is the principle behind rational drug design in identifying lead compounds?

Utilizes the understanding of a target's structure and function to design compounds that interact with it in a desired manner, potentially leading to the identification of lead compounds.

Can drugs target proteins other than enzymes and receptors?

Yes, drugs can also target other proteins involved in various cellular processes, such as transport proteins, ion channels, structural proteins, or proteins involved in gene expression and regulation.

Do drugs interact with DNA and RNA?

Yes, some drugs can target nucleic acids like DNA or RNA. They may interact with specific sequences to modulate gene expression, inhibit replication, or interfere with cellular processes.