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3.1.9: Discovery and development of drugs

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Traditional drug sources:

Digitalis (heart drug): from foxgloves.

Aspirin (painkiller): from willow.

Penicillin (antibiotic): discovered by Alexander Fleming from the Penicillium mould.

Most new drugs are synthesised by chemists (though may start from a plant chemical).

Testing new drugs — three criteria: toxicity (is it safe?), efficacy (does it work?), dose (how much to give?).

Preclinical testing: in laboratory using cells, tissues and live animals; checks for toxicity.

Clinical trials:

Stage 1: very low doses given to healthy volunteers to check safety.

Stage 2: given to patients to check efficacy and find optimum dose.

Double-blind trial: some patients receive a placebo (inactive substance); neither doctors nor patients know who has the real drug.

Results published only after peer review (scrutiny by other scientists).

Common exam mistakes

Aspirin comes from willow (not foxgloves); digitalis comes from foxgloves (not willow) — these are frequently swapped.

Toxicity and side effects are essentially the same thing — cannot be counted as two separate points.

Efficacy = effectiveness — do NOT say 'efficiency', which has a different meaning.

In double-blind trials, neither the doctor NOR the patient knows who receives the real drug — both are 'blind'.

Peer review is the checking of results by other independent scientists before publication — not just having the trial repeated.

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