Drugs bind to specific targets, activating (stimulating) or inactivating (blocking)

their functions and altering their biologic responses.

A RECEPTORS Ion channel

Direct opening/closing
Enzyme
activation/inhibition
Agonist/ Transduction Ion channel
inverse mechanisms modulation
agonist DNA
transcription

No effect
Antagonist
Endogenous mediators blocked

B ION CHANNELS
Blockers Permeation
blocked
Increased or
Modulators decreased
opening probability

C ENZYMES
Normal reaction
Inhibitor
inhibited

False Abnormal
substrate metabolite produced

Prodrug Active drug produced

D TRANSPORTERS

Normal
transport

or Transport
Inhibitor
blocked

False Abnormal compound

substrate accumulated

Agonist/substrate Abnormal product

Antagonist/inhibitor Prodrug
Drugs target macromolecules to modify ongoing cellular processes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand,
get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either
mimicking the action of endogenous ligands or blocking the receptor activity to bring about a
modified response.
A second class of drug targets includes the ion channels that facilitate the ion movement across
the cell and help in neurotransmission or muscle contraction. Ion channels can be ligand-gated,
whose opening and closing are controlled by an external ligand, or voltage-gated, controlled by
changing membrane potential. Drugs target ion channels either by binding to the ligand binding
site or the allosteric site, or by directly blocking the channel pore. Sedatives such as
benzodiazepines bind at the allosteric site of the GABA receptor, a ligand-gated ion channel, and
enhance the binding of GABA to the ligand-binding site. In contrast, vasodilators such as
dihydropyridine block L-type calcium channels in cardiac muscle cells.
A third class of drug targets includes transporters. Transporters help move small lipid-insoluble
molecules and other metabolites across the membrane. Many available drugs block the
functioning of these transporters. For example, diuretics such as furosemide and
hydrochlorothiazide, block kidney tubule transporters to prevent sodium reabsorption.
Several other drugs target enzymes, or other biological catalysts, that carry out biochemical
reactions inside a cell. Some drugs act as substrate analogs that may bind reversibly or irreversibly
to inhibit enzyme activity. Others work as false substrates, which bind the enzyme to allow the
biochemical reaction but form an abnormal end product. Examples include fluorouracil, an
anticancer drug that replaces uracil during purine biosynthesis, blocking DNA biosynthesis and
inhibiting cell division.
In addition to these targets, certain drugs, such as colchicine, interact with tubulin, a structural
protein, and are often used to treat arthritic gout. Others prevent bacterial invasion or cancer cell
proliferation by targeting bacterial cell wall proteins, DNA, and other proteins.