Summary Molecular Therapy 2020-2021 – Gül Turan
Week 1
Personalized healthcare includes many developments. The main principle is having drugs that is
beneficial and NOT toxic for individuals. This is mainly stated as such; having the right drug at the
right time for the right patient at a right dose.
Example personalized medicine in melanoma
B-RAFv600E mutation is part of the ERK signalling cascade which is responsible for proliferation.
B-RAFv600E cells always grow and become cancer cells. RAF inhibitors block pathway and block cell
growth and inhibit cancers that have a B-RAFv600E mutation.
Phase 1 – testing drug in small group of patients
Phase 2 – scaling up confirmative phase (POC)
Phase 3 – scaling up more
Phase 4 – introduce on the market and monitor how drug behaves after the market introduction
Pharmacokinetics - movement of the drugs through the body. What does the patient do with the
drugs.
Pharmacodynamics: The body’s biological response to the drugs. What does the drug do in the
patient.
There are three pharmacological phases in pharmacotherapy
1. Exposition - behaviour of a substance in the environment, changes in the application form,
available for uptake
2. Toxicokinetic - Absorption, Distribution, Metabolism (toxification, detoxification), Excretion.
(ADME)
3. Toxicodynamic - interactions with receptors or other (macro)molecules at the site of the
operation.
Drugs can be agonists or antagonists
Agonist: When it binds to the receptor it leads to a function (signal transduction)
Antagonist: When it binds to the receptor it inhibits the function so no effect will occur
Week 1
Personalized healthcare includes many developments. The main principle is having drugs that is
beneficial and NOT toxic for individuals. This is mainly stated as such; having the right drug at the
right time for the right patient at a right dose.
Example personalized medicine in melanoma
B-RAFv600E mutation is part of the ERK signalling cascade which is responsible for proliferation.
B-RAFv600E cells always grow and become cancer cells. RAF inhibitors block pathway and block cell
growth and inhibit cancers that have a B-RAFv600E mutation.
Phase 1 – testing drug in small group of patients
Phase 2 – scaling up confirmative phase (POC)
Phase 3 – scaling up more
Phase 4 – introduce on the market and monitor how drug behaves after the market introduction
Pharmacokinetics - movement of the drugs through the body. What does the patient do with the
drugs.
Pharmacodynamics: The body’s biological response to the drugs. What does the drug do in the
patient.
There are three pharmacological phases in pharmacotherapy
1. Exposition - behaviour of a substance in the environment, changes in the application form,
available for uptake
2. Toxicokinetic - Absorption, Distribution, Metabolism (toxification, detoxification), Excretion.
(ADME)
3. Toxicodynamic - interactions with receptors or other (macro)molecules at the site of the
operation.
Drugs can be agonists or antagonists
Agonist: When it binds to the receptor it leads to a function (signal transduction)
Antagonist: When it binds to the receptor it inhibits the function so no effect will occur
