1. THE QUEST FOR THE CURE

2. THE DRUG DISCOVERY CRISIS
Are we running out of drugs??

3. HOW DO DRUGS WORK
Most drugs (their active principle) are small molecules
OMEPRAZOL
DIAZEPAM
IBUPROFEN
PROZAC
ATORVASTATIN

4. DRUGS OF ABUSE
C
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c
a
i
n
e
Morphine
Cannabinol (THC)
Heroine (Bayer, 1874)
The active principle is also a small molecule
Addiction as a side effect makes them not useful

5. HOW DO DRUGS WORK
Protein Drugs work by binding to specific proteins (enzymes)
and modifying their activity
The selectivity of a drug in provided by the 3D
structure of the target protein
Antibiotics and antivirals are drugs that inhibit bacterial and viral proteins,
while leave ours untouched
Besides the large number of drugs available,
only 2% of the human proteins are known to targeted by the drugs

6. HOW NEW DRUGS ARE
DISCOVERED
In the past:
• by identifying the active ingredient from traditional
remedies (plant extracts…etc. )
• by serendipitous discovery (e.g. penicillin,
benzodiazepines)
Nowadays:
• Large scale testing of chemical collections
• Rational design

7. HOW NEW DRUGS ARE
DISCOVERED
Basic Science Screening thousand
of compounds
Target Development of an assay
candidate for the desired activity Preclinical & clinical studies
Rational design based on :
• Structure of the target.
• Modification of known drugs

8. STAGES IN THE DRUG DISCOVERY PROCESS
Target selection
Food and Drug Administration (FDA) in the United States (or comparable agency in other countries)
The development of a new drug can take several decades and billion dollars invested
Lombardino & Lowe. Nature Reviews Drug Discovery (2004) 3, 853-862

9. THE DRUG DISCOVERY CRISIS
The future
• The better understanding of human
biology will offer new candidates to find drugs
Are we running out of drugs?? (We must support basic research!!!)
• New technologies for the rational design of drugs
are in development. They will reduce costs and
will provide new medicines.
• There is little knowledge yet about how
drugs interact.
• Gene therapy methods will join the “small molecule”
toolbox once their safety is assured.