Goodman and Gilman's The
Pharmacological Basis of Therapeutics
14th Edition
Author(s)Laurence Brunton; Bjorn Knollmann
Print ISBN: 9781264258079
,Question 1
A research team investigates a medicinal plant traditionally used to
relieve pain. After preparing an extract, the investigators isolate a
purified compound that reproduces the extract’s analgesic activity in a
standardized assay. What is the most appropriate interpretation of this
compound?
,A. It is a validated therapeutic drug
B. It is a lead compound that may undergo further optimization
C. It is automatically the safest component of the plant
D. It has established clinical efficacy because it is naturally derived
Correct Answer:
B. It is a lead compound that may undergo further optimization
Rationale:
B is correct because a biologically active, isolated compound can serve
as a lead for further pharmacological and medicinal-chemistry studies.
Lead status does not mean that clinical efficacy, safety, or suitable
pharmacokinetics have already been established.
A is incorrect because identification of biological activity does not
establish clinical efficacy or regulatory approval.
C is incorrect because naturally occurring compounds can have
substantial toxicity and adverse effects.
D is incorrect because natural origin does not guarantee efficacy or
safety in humans.
Question 2
Which step most directly converts a crude medicinal-plant preparation
into information about a specific chemical entity responsible for
biological activity?
A. Regulatory approval
B. Isolation and structural characterization of active constituents
C. Phase III clinical testing
D. Postmarketing surveillance
, Correct Answer:
B. Isolation and structural characterization of active constituents
Rationale:
B is correct because separating active constituents and determining
their chemical structures allows investigators to relate biological activity
to a defined molecular entity.
A is incorrect because regulatory approval occurs much later and
requires extensive evidence.
C is incorrect because phase III studies evaluate clinical efficacy and
safety after substantial preclinical development.
D is incorrect because postmarketing surveillance occurs after a product
reaches clinical use.
Question 3
In medicinal chemistry, a compound that demonstrates useful biological
activity but still requires optimization of its potency, selectivity,
pharmacokinetics, or safety is best described as a:
A. Lead compound
B. Placebo
C. Biomarker
D. Formulation excipient
Correct Answer:
A. Lead compound
Rationale:
A is correct because a lead compound has properties suggesting that it
can serve as a starting point for chemical optimization toward a drug
candidate.