Pharmacology

Glossary of Terms

Absorption
The uptake of a drug from its site of administration into the bloodstream.

Active transport
The movement of a molecule against its concentration gradient across a membrane, with a
concomitant expenditure of energy.

Adverse drug effect

A harmful or toxic effect produced by a drug in a patient.

Affinity
A measure of the strength of drug-receptor binding.

Agonist
An endogenous or exogenous ligand that binds to and stimulates a receptor to produce a
molecular response.

Anabolic reaction
A reaction involving synthesis of new molecules, generally requiring energy.

Analgesics
Drugs that relieve pain.

Anaphylactic reaction
A severe allergic reaction that can be life-threatening.

Antagonist
An endogenous or exogenous molecule that binds to and blocks a receptor to inhibit a
molecular response.

Antibody
A glycoprotein that plays an important role in the immune response by binding to and
targeting specific antigens for removal from the body.

Anticoagulants
Drugs that prevent or reduce blood clotting.

Pharmacology
Glossary of Terms
Antiemetics
A class of drugs that can be used in the treatment or prevention of nausea and/or vomiting

Antigen
A substance, generally foreign to the body, that induces an immune response.

Area under the curve (AUC)

When the concentration of drug in the plasma is plotted over time on a graph, the AUC is
used as a measure of the total exposure of the body to a drug.

Astrocytes
Specialized glial cells in the brain that contribute to the integrity of the blood-brain barrier.

Basal activity
The molecular response produced by a receptor in the absence of a bound ligand, also
referred to as constitutive activity.

Bile
A fluid produced by the liver which aids digestion of fats in the small intestine and helps to
remove toxins from the body.

Binding curve
A graph representing the percentage of receptor occupancy, or the percentage of receptors
bound by a drug, plotted against the concentration of drug at the receptor.

Bioavailability
The fraction of administered drug that reaches the systemic circulation.

Bioavailable
The amount of drug that is available in the systemic circulation.

Biologics
Therapeutics that are large in size (biological polymers), have complex structures, and are
derived from natural sources.

Blood-brain barrier
A protective membrane barrier between the blood and the brain that consists of endothelial
cells joined by tight junctions and surrounded by astrocytes.

Bolus dose
A single dose of a drug.

Bronchodilator
A drug used in the treatment of asthma and other lung disorders. It acts by relaxing bronchial
muscle, resulting in expansion of the bronchial air passages and increased airflow to
the lungs.

C50
A concentration corresponding to half the maximal concentration of drug in the bloodstream.

Carcinogen
A drug or substance that can cause cancer.

Carcinogenesis
A process by which normal cells in the body become cancerous.

Pharmacology
Glossary of Terms
Catabolic reaction
A reaction involving breakdown of molecules, generally releasing energy.

Cell-based therapy
A therapy that involves introducing live cells into a patient. These cells can be obtained from
the patient or another individual, and may be modified genetically or developmentally
before use.

Cellular protein
A protein found within a cell or cell membrane such as a receptor, an enzyme, an ion channel,
or a transporter.

Cerebrospinal fluid (CSF)

The clear liquid surrounding the brain and spinal cord.

Characterization (of a compound)

The process of determining the biological, chemical, toxic, and pharmacological properties
of a compound during drug development.

Chemical name
A name for a drug that describes its molecular structure.

Clearance
The volume of plasma cleared of a drug in a unit of time.

Clinical drug development

Studies done in a clinical setting that involve testing drugs in humans.

Cmax
The maximum concentration of drug achieved in the bloodstream.

Competitive antagonist
An antagonist that binds to a receptor, thereby inhibiting the binding of an agonist to that
receptor and preventing a molecular response from being produced by the agonist.

Compound-centered drug discovery

Drug discovery that begins by identifying a compound.

Concentration gradient
The difference in concentration of a drug across a cell membrane.

Conjugation reaction
A reaction in which a small, polar, endogenous molecule is joined to another molecule.

Continuous infusion
Administration of a drug into a blood vessel over a prolonged period of time.

Cytochrome P450 enzymes (CYP enzymes)

A superfamily of related but distinct enzymes, found primarily in the liver, that are involved in
phase 1 reactions of drug metabolism.

Dalton (Da)
A standard unit of mass equivalent to 1 gram per mole.

Distribution
Movement of a drug from the bloodstream into body tissues, including the tissue(s)
containing the drug’s molecular target.

Pharmacology
Glossary of Terms
Dose-response curve
A graph representing the magnitude of a biological response plotted against the
concentration of drug at the receptor.

Double-blind trial
A design of a clinical trial where neither the patient nor the clinician knows what treatment is
being administered to the patient.

Downstream signaling
The cellular signaling produced or inhibited as a result of a drug binding to its
molecular target.

Drug candidate
A compound identified during drug discovery or design that enters preclinical drug
development to determine whether it could be effective in treating a particular
disease or disorder.

Drug design
Design and synthesis of a new compound that may have biological activity.

Drug discovery
Identification of a natural or synthetic compound that may have biological activity.

Drug-disease interactions
An interaction between a drug and a patient’s disease state that can result in an adverse
drug effect.

Drug-drug interactions
An interaction between two drugs that can result in an adverse drug effect.

Drug-environment interactions
An interaction between a drug and a food, beverage, herbal or dietary supplement, or the
patient’s external environment that can result in an adverse drug effect.

Drug modalities
Types of drugs, often grouped according to their molecular size and chemical structure.

Drug-receptor binding
The binding of a drug to a receptor to form a drug–receptor complex.

Drug toxicity
A harmful or toxic effect caused by a drug in a patient.

EC50
The concentration of drug required to produce 50% of the maximal response, used as a
measure of potency.

ED50
The dose of drug that is effective in 50% of a population, where ED stands for effective dose.

Efficacy
The maximal response produced by a drug when it binds to a molecular target.

Emax
The maximal response produced by a drug when it binds to a molecular target, used as
a measure of efficacy.

Pharmacology
Glossary of Terms
Enteral routes of administration
Routes of administration that involve the GI tract.

Enzyme
A protein that catalyzes (accelerates) a biochemical reaction.

Equilibrium dissociation constant (Kd)

The concentration of drug at which the drug occupies 50% of its receptors.

Excretion
The removal of a drug from the body.

Exponential
An increase or decrease of a quantity at a steady rate that is proportional to its current value.

Extracellular fluid
The fluid outside the cells of the body. This fluid is commonly divided into plasma, lymph,
interstitial fluid, and transcellular fluid.

Facilitated diffusion
Movement of a drug across a cell membrane typically via a protein channel or pore, from an
area of high concentration to an area of low concentration.

Fat-soluble molecules
Molecules that dissolve well in fats.

Fick’s Law
A law describing movement of molecules across a membrane, which describes how the
rate of diffusion depends on the surface area, the diffusion distance, and the concentration
gradient across a cell membrane.

First-order kinetics
The elimination of a drug at a rate that is proportional to the plasma concentration of
the drug.

First-pass metabolism
Metabolism of a drug in the liver before it enters the systemic circulation.

Food and Drug Administration (FDA)

A federal agency of the United States Department of Health and Human Services that has a
very broad range of regulatory responsibilities, including ensuring that drugs are
safe and effective.

Full agonist
An agonist that binds to and stimulates a receptor to produce the maximal molecular
response the receptor is capable of producing.

Gastrointestinal tract (GI tract)

The digestive tract, divided into the mouth, esophagus, stomach, small intestine, large
intestine, and rectum.

Gene-based therapy
A therapy that involves introducing genetic material, in the form of DNA or RNA, into cells of
a patient.

Pharmacology
Glossary of Terms
Generic name
The name given to a drug by an official body.

Genetic variation
Genetic differences in the DNA sequence among individuals in a population.

GI effects
In pharmacology, this refers to any adverse effects a drug has on the GI tract such as
nausea, vomiting, stomach pain, or loss of appetite.

GI toxicity
Toxicity to the GI tract.

Glial cells
Cells that provide support and insulation for neurons in the central and peripheral
nervous system.

Glomerular filtration
The process of filtration of a drug or metabolite from the plasma in the glomerular capillaries
into the filtrate in the tubule of the nephron.

G protein-coupled receptor
The largest superfamily of receptors, these molecules reside in the cell membrane and signal
via activation of G proteins.

Half-life (t1/2)
The amount of time it takes for the concentration of a drug in the plasma to decrease by half.

Hepatotoxicity
Toxicity to the liver.

Hit
A compound identified during screening, in target-centered drug discovery, which shows
selectivity for a specific target or successfully alters the activity of this target.

Hydrolysis reaction
A chemical reaction where a molecule of water is incorporated into another molecule, which
is consequently split into two parts.

Hydrophilic molecules
Molecules that mix well in water (water-soluble).

Hydrophobic molecules
Molecules that mix well in fats (fat-soluble).

Hydroxyl group
In a molecule, a negatively charged group composed of a bonded oxygen and
hydrogen atom (OH).

IC50
The concentration of an antagonist required to reduce the maximal response by 50%.

Immunological reaction
In drug toxicity, this is an adverse drug effect mediated by the immune system.

Pharmacology
Glossary of Terms
Individual variation
Factors that can lead to pharmacokinetic or pharmacodynamic changes between individuals
or in an individual over time, such as age, genetics, weight, sex, health status, and pregnancy.

Inhalation administration
Administration of a drug within the respiratory tract by inhaling via the nose and/or mouth.

Inhibitory constant (Ki)

The concentration of an antagonist required to bind 50% of receptors.

Intended molecular target

The molecular target in the body that a given drug is intended to alter the activity of, in order
to produce a therapeutic effect.

Interstitial fluid
The fluid that surrounds the tissues in the body.

Interstitial nephritis
Inflammation and swelling between the tubules of the kidney.

Intracellular fluid
The fluid within the cells of the body.

Intramuscular administration
Administration of a drug within a muscle.

Intrathecal administration
Administration of a drug within the cerebrospinal fluid.

Intravenous (IV) administration

Administration of a drug within or into a vein or veins.

Inverse agonist
An agonist that binds to and inhibits the basal activity of the receptor; some molecules of
this type also inhibit full agonists or partial agonists from binding to the receptor and thereby
block a stimulatory molecular response.

Investigational New Drug application (IND)

An application made to the relevant regulatory authority, such as the FDA, to request
approval to move a drug candidate from preclinical drug development into clinical
drug development.

In vitro test
A test performed in an artificial environment outside the living organism, such as a test tube
or petri dish.

Ion channel
A protein that, upon activation, acts as a selective or nonselective pore in
the cell membrane.

Ionized
Having an electrical charge (charged). Ionized drugs are often more water-soluble.

Kinase-linked receptor
Receptors in the cell membrane that have an intracellular domain with enzymatic
phosphorylation activity.

Pharmacology
Glossary of Terms
LD50
The dose of drug that is lethal in 50% of a population, where LD stands for lethal dose.

Lead compound
The most promising compound, or hit, that is revealed after the characterization and
chemical modification processes in target-centered drug discovery. This compound is moved
forward in preclinical drug development.

Lethal dose
A dose of drug that is lethal.

Ligand-gated ion channel

Ion channels that ligands bind to in order to allow the passage of ions across
the cell membrane.

Linear scale
An axis on a graph that has grid lines increasing sequentially with equal divisions for
equal values.

Loading dose
A high drug dose given at the start of a course of treatment to rapidly increase the
concentration of the drug in the plasma to the desired steady-state level.

Log scale
An axis on a graph where each grid line on the axis is a multiple of the preceding grid line,
most often a multiple of 10, used to represent wide data ranges.

Lymphatic system
A component of the circulatory system made up of vessels that move infection-fighting white
blood cells around the body in a fluid referred to as lymph.

Margin of safety
The ratio between the effective dose of a drug and the dose of drug that causes adverse
drug effects, or toxicity.

Mechanism of action
The biochemical and physiological interactions by which a drug exerts a therapeutic effect.

Metabolism
The chemical modification or breakdown of a drug in the body, also commonly referred to
as biotransformation.

Molecular target
A target in the body, often a cellular protein, that a drug can bind to and alter the activity of,
in order to produce a therapeutic effect.

Monoclonal antibodies
Antibodies produced by a single clone of cells that can be used to target specific antigens or
receptors in the body.

Myasthenia gravis
A chronic neuromuscular disease that leads to weakness of the skeletal muscle.

Myocardial infarction
Acute cardiac damage caused by the blockage of blood flow to the heart muscle, also known
as a heart attack.

Pharmacology
Glossary of Terms
Myopathy
A disorder of the skeletal muscle which can lead to dysfunction or impairment of the muscle.

Negative efficacy
An efficacy value less than 0, meaning that the drug decreases the activity of a
molecular target.

Nephrotoxicity
Toxicity to the kidneys.

Neurochemical
A small organic chemical that acts on the central nervous system.

Neurotoxicity
Toxicity to the central or peripheral nervous system.

Neutral efficacy
An efficacy value of 0, meaning that the drug does not alter the activity of a
molecular target.

New Drug Application (NDA)

An application made to the relevant regulatory authority, such as the FDA, to request
approval to move an investigational drug from phase III clinical trials onto the market, i.e., to
be available for use by patients as a drug.

Non-competitive antagonist
An antagonist that binds to an alternative site on a receptor and thereby inhibits a molecular
response from being produced by a bound agonist.

Non-ionized
Having no electrical charge (uncharged). Non-ionized drugs are often more fat-soluble.

Nonpolar molecules
Molecules that have uniformly distributed electrons. Nonpolar molecules dissolve freely in
membrane lipids.

Nuclear receptor
Intracellular receptors that upon activation, can enter the nucleus and interact directly with
DNA to regulate gene transcription.

Ocular
Relating to the eye.

Off-target mechanisms of toxicity

Drug toxicity caused by a drug binding to an unintended receptor in the intended tissue or in
an unintended tissue.

Onset of action
The length of time from administration of a drug to the initiation of its therapeutic effect.

On-target mechanisms of toxicity

Drug toxicity caused by a drug binding to its intended receptor in the intended tissue or in an
unintended tissue.

Opioids
A class of legal and illegal drugs that are used clinically as powerful pain relievers, and which
act on the opioid receptors, mainly in the central nervous system.

Pharmacology
Glossary of Terms
Optimization (of a compound)
A process that involves modification of a lead compound to alter undesirable properties of
that compound with the objective of creating a drug candidate suitable for preclinical
drug development.

Oral administration
Administration of a drug to or by way of the mouth.

Organ toxicity
Toxicity to a specific organ in the body.

Oxidation reaction
A chemical reaction in which a molecule loses electrons.

Parenteral routes of administration

The routes of administration that bypass the GI tract, such as injection and infusion.

Partial agonist
An agonist that binds to and stimulates a receptor to produce a submaximal
molecular response.

Partition
The equilibrium distribution of a drug into the various compartments of the body, including
the vascular, interstitial, intracellular, transcellular, and fat compartments.

Passive diffusion
Movement of a drug or metabolite, typically across a cell membrane, from a region of high
concentration to one of low concentration.

Peritoneal cavity
The potential space within the sac that surrounds the abdominal organs.

Pharmacovigilance trials
Clinical trials performed to detect, assess, understand, and prevent adverse effects of
marketed drugs. This term is used interchangeably with phase IV clinical trials and
post-marketing surveillance.

Phase 1 reactions
Catabolic reactions of drug metabolism in the liver, such as oxidation, reduction, and
hydrolysis. These reactions are often mediated by cytochrome P450 enzymes.

Phase 2 reactions
Anabolic reactions of drug metabolism in the liver, such as conjugation.

Phase I clinical trials

The first phase of clinical drug development to determine the ADME and safety parameters
of an investigational drug, commonly performed in healthy volunteers.

Phase II clinical trials

The second phase of clinical drug development to determine the efficacy and dosing
parameters of an investigational drug in patients with the condition of interest, often in
comparison to a placebo.

Phase III clinical trials

The third phase of clinical drug development to determine the efficacy and tolerability of
an investigational drug in patients with the condition of interest, often in comparison to a
standard of care medication for the condition of interest.

Pharmacology
Glossary of Terms
Phase IV clinical trials
The fourth and final phase of clinical drug development to determine the long-term efficacy
and safety of a drug after it is available on the market for patient use.

Physiological response
A response in the body that results from normal changes in homeostatic pathways, such as
those regulating heart rate and blood pressure.

Placebo
An inactive substance that is designed to look exactly like a real drug, commonly made of a
starch or sugar, and does not contain an active drug.

Plasma
The liquid portion of the blood.

Polar molecules
Molecules that have unevenly distributed electrons, which do not dissolve freely in
membrane lipids.

Portal circulation
The circulation of blood from the abdominal organs (small intestine, colon, pancreas, and
spleen) to the liver.

Positive efficacy
An efficacy value greater than 0, meaning that the drug increases the activity of a
molecular target.

Potency
The concentration of drug that is required to produce 50% of the maximal response.

Preclinical drug development

Drug studies done in a research laboratory setting that most often involve testing drugs at
the molecular and cellular level, or at the organismal level in animal experiments.

Prodrug
A drug that is delivered in an inactive form and is activated by metabolism.

Protein-based therapy
A therapy that involves the introduction of functional, novel, or modified proteins into a
patient that can be used to replace missing or defective proteins, to alter the activity of an
existing pathway, to provide a novel function, or to target a specific molecule for destruction,
such as an antigen.

Prothrombotic state
A state associated with an increased tendency to blood clotting, also referred to as a
hypercoagulable state.

Psychomotor activity
An activity that involves conscious coordination of motor skills.

Psychostimulant
A drug that has stimulant properties and increases psychomotor activity.

Rate of elimination
The fraction of a drug that is eliminated per unit of time. Often refers to the elimination of a
drug from the bloodstream.

Pharmacology
Glossary of Terms
Reabsorption
The process of a drug or metabolite being transported from the tubule of the nephron into
the plasma in the peritubular capillaries.

Receptor
A macromolecule to which agonists and antagonists can bind and thereby produce a
physiological or pharmacological effect.

Receptor occupancy
The fraction of receptors that are bound by a drug, often expressed as a percentage.

Rectal administration
Administration of a drug to the rectum.

Reduction reaction
A chemical reaction in which a molecule gains electrons.

Route of administration
The method by which a drug is introduced into the body.

Second messenger-regulated ion channel

A type of ion channel that is regulated by signaling downstream of G protein-coupled
receptors or other receptors.

Selective Estrogen Receptor Modulators (SERMs)

A group of drugs that bind to and alter the activity of the estrogen receptor.

Selective Serotonin Reuptake Inhibitors (SSRIs)

A group of drugs that block the reuptake of serotonin in the brain and can be used in the
treatment of depression.

Selectivity
A measure of the fraction of a drug that binds to its intended molecular target compared to
other, unintended molecular targets.

Semi-log plot
A graph that has one axis with a linear scale, most often the y-axis, and another axis with a
logarithmic (log) scale, most often the x-axis.

Semi-synthetic drug
A drug based on a natural product that is then chemically modified.

Single-blind trial
A design of a clinical trial where the patient does not know but the clinician knows what
treatment is being administered to the patient.

Small molecule drugs

Drugs that have a molecular mass of 900 Da or less, have simple structures, and are generally
chemically synthesized.

Steady state
Relating to drug dosing, this occurs when the rate of absorption of a drug into the plasma
equals the rate of clearance of the drug from the plasma.

Subcutaneous administration
Administration of a drug beneath the skin.

Pharmacology
Glossary of Terms
Sublingual administration
Administration of a drug beneath the tongue.

Subtherapeutic dosing
Occurs when the concentration of drug in the plasma is insufficient to produce a
therapeutic effect.

Sympathomimetic drug
A drug that mimics endogenous agents that act on the targets of the sympathetic nervous
system, which is responsible for regulating many physiological parameters, such as heart rate
and blood pressure.

Systemic circulation
A part of the cardiovascular system that includes the circulation of blood to and from
the heart.

Target-centered drug discovery

Drug discovery that begins by identifying a target molecule.

T cell
A type of white blood cell involved in the immune response.

TD50
The dose of drug that is toxic in 50% of a population, where TD stands for toxic dose.

Teratogen
A drug or substance that can cause teratogenesis.

Teratogenesis
The induction of structural defects in the fetus, which are also called fetal abnormalities.

Therapeutic effect
The desired response initiated by a drug or treatment.

Therapeutic index (TI)

The ratio between the TD50 and the ED50, which is also used as an indication of the margin of
safety of a drug.

Thromboemboli
Blood clots formed in a blood vessel that can break away and travel through the body in the
bloodstream, potentially lodging in and blocking another blood vessel.

Tight junctions
Cell-cell junctions found between adjacent epithelial cells.

tmax
The amount of time it takes to reach the maximum concentration of drug in the plasma
after administration.

Topical administration
Application of a drug to an epithelial surface in the body.

Total body clearance

The volume of plasma cleared of a drug in a unit of time by renal clearance, hepatic
clearance, and all other clearance mechanisms.

Pharmacology
Glossary of Terms
Toxic dose
A dose of drug that is toxic.

Toxic dosing
Administration of a drug resulting in excessive plasma concentrations that lead to
toxic effects.

Trade name
A legally registered trademark of a compounding or formulation of a drug by
a pharmaceutical company.

Transcellular fluid
The fluid formed from the secretions of epithelial cells, found within epithelial-lined spaces
(e.g. the synovial fluid in joints, the amniotic fluid in the womb).

Transdermal administration
Administration of a drug through the skin to the systemic circulation.

Transferases
A class of enzymes that catalyze conjugation reactions.

Transporter
A carrier protein found in the cell membrane that assists in the movement of molecules
across the membrane.

Tubular secretion
The process of active drug or metabolite secretion out of the plasma from the peritubular
capillaries into the tubule of the nephron.

Unintended molecular target

A molecular target in the body that a given drug is not intended to bind to or
alter the activity of.

Vaccine
A substance or biological preparation delivered to a patient to improve immunity to
a specific disease.

Viral vector
A virus modified so that it can deliver nucleic acids into a cell but it cannot cause disease.

Voltage-gated ion channel

A type of ion channel that is activated by local changes in electrical membrane potential.

Volume of distribution (Vd)

The apparent volume of body fluid required to contain the total amount of an administered
drug at the steady-state drug concentration observed in the plasma.

Water-soluble molecules
Molecules that dissolve well in water.

Weak acid
A molecule that donates a hydrogen ion when it dissociates, but which does not dissociate
completely at physiological pH levels.

Pharmacology
Glossary of Terms
Weak base
A molecule that accepts a hydrogen ion when it dissociates, but which does not dissociate
completely at physiological pH levels.

Zero-order kinetics
The elimination of a drug at a rate that is independent of the plasma concentration of
the drug.

Copyright © The President and Fellows of Harvard College

Pharmacology
Glossary of Terms