Molecular Therapy: Summary Exam
Tutorial 1. Pharmacology: pharmacodynamics and pharmaco -
kinetics
− Pharmacology = the science that is concerned with the (beneficial) uses, effects and
modes of action of chemicals on the function of living systems.
− Pharmacotherapy = the use of pharmacology to cure a patient.
o Evidence-based pharmacotherapy = it does not matter how it works, as long as
it works.
o Rational pharmacotherapy = mechanism-based pharmacotherapy (pharmaco-
kinetics & pharmacodynamics).
− Pharmacokinetics = what does the patient do with the drug (e.g. absorption,
metabolism).
− Pharmacodynamics = what does the drug do with the patient (bind to receptors etc.)
There are different pharmacological phases, after the dose is given until the effect is seen:
− Exposition phase = the behaviour of a substance in the environment, changes in the
application form, availability for uptake.
− Toxicokinetic phase = absorption, distribution, biotransformation (toxification,
detoxification), excretion.
− Toxicodynamic phase = interactions with receptors or other (macro) molecules at the site
of the operation.
Agonist = a drug that works on a receptor (on molecular scale).
Antagonist = a molecule that can also bind to the same receptor, but it does not give any effect.
It blocks the binding of the agonist.
The toxicity of a substance depends on the dose: medicines have beneficial effects, but can
have toxic effects in too large doses.
,The molecule (drug) can activate different kind of pathways via different types of receptors.
➔ In the case of a painkiller, it should work as fast as possible, favouring ligand-gated ion
channels or G-protein-coupled receptors.
Ion channels, enzymes and transporters can also act like receptors. They are influenced by other
components, with a change in effect as a consequence.
,The amount and the types of bonds present in a molecule influences the kinetics and dynamics.
− Some specific bonds are stronger than other bonds, making a more stable molecule,
which is less easy degradable.
− Most often, multiple types of bonds are present in a molecule.
− A covalent binding is not reversible.
Molecules and drugs are transported across cell membranes:
− Transport proteins use gradients for transport.
− The Na+, K+, ATPase uses ATP to keep the gradient intact.
, Different curves can be made regarding the concentration of a drug and its binding state.
− Fractional occupancy = how much of the molecule is bound to a receptor.
− KA = concentration point where half of the receptors have a ligand bound, this says
something about the affinity of the drug to the receptor.
o The lower the KA, the higher the affinity of the drug to the receptor.
− The binding state of the receptor cannot always be seen / measured. Physiological
changes can be measured (e.g. blood pressure), which is the response (%max).
o KA → fractional occupancy.
o EC50 → response.
Tutorial 1. Pharmacology: pharmacodynamics and pharmaco -
kinetics
− Pharmacology = the science that is concerned with the (beneficial) uses, effects and
modes of action of chemicals on the function of living systems.
− Pharmacotherapy = the use of pharmacology to cure a patient.
o Evidence-based pharmacotherapy = it does not matter how it works, as long as
it works.
o Rational pharmacotherapy = mechanism-based pharmacotherapy (pharmaco-
kinetics & pharmacodynamics).
− Pharmacokinetics = what does the patient do with the drug (e.g. absorption,
metabolism).
− Pharmacodynamics = what does the drug do with the patient (bind to receptors etc.)
There are different pharmacological phases, after the dose is given until the effect is seen:
− Exposition phase = the behaviour of a substance in the environment, changes in the
application form, availability for uptake.
− Toxicokinetic phase = absorption, distribution, biotransformation (toxification,
detoxification), excretion.
− Toxicodynamic phase = interactions with receptors or other (macro) molecules at the site
of the operation.
Agonist = a drug that works on a receptor (on molecular scale).
Antagonist = a molecule that can also bind to the same receptor, but it does not give any effect.
It blocks the binding of the agonist.
The toxicity of a substance depends on the dose: medicines have beneficial effects, but can
have toxic effects in too large doses.
,The molecule (drug) can activate different kind of pathways via different types of receptors.
➔ In the case of a painkiller, it should work as fast as possible, favouring ligand-gated ion
channels or G-protein-coupled receptors.
Ion channels, enzymes and transporters can also act like receptors. They are influenced by other
components, with a change in effect as a consequence.
,The amount and the types of bonds present in a molecule influences the kinetics and dynamics.
− Some specific bonds are stronger than other bonds, making a more stable molecule,
which is less easy degradable.
− Most often, multiple types of bonds are present in a molecule.
− A covalent binding is not reversible.
Molecules and drugs are transported across cell membranes:
− Transport proteins use gradients for transport.
− The Na+, K+, ATPase uses ATP to keep the gradient intact.
, Different curves can be made regarding the concentration of a drug and its binding state.
− Fractional occupancy = how much of the molecule is bound to a receptor.
− KA = concentration point where half of the receptors have a ligand bound, this says
something about the affinity of the drug to the receptor.
o The lower the KA, the higher the affinity of the drug to the receptor.
− The binding state of the receptor cannot always be seen / measured. Physiological
changes can be measured (e.g. blood pressure), which is the response (%max).
o KA → fractional occupancy.
o EC50 → response.
