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3.1.9: Discovery and development of drugs

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Traditional drug sources:

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Digitalis (heart drug): from foxgloves.

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Aspirin (painkiller): from willow.

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

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

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

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

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Clinical trials:

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

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

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

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

Common exam mistakes

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

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

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

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

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

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