Identify TWO different diseases/disorders for which named drugs in use in the clinic target enzymes which regulate the levels of endogenous steroids. For EACH disease,

PHAR4007E1
SECTION A
(60 marks)

1. Steroids are endogenous hydrophobic hormones generated from cholesterol.

Identify similarities and differences between the multiple molecular targets of
endogenous steroids in humans. In your answer, focus on the:

a) Primary sequence comparisons of the molecular targets for the endogenous steroids

[3 marks]

b) Signalling characteristics of the molecular targets for the endogenous steroids

[4 marks]

c) Tissue locations of the molecular targets for the endogenous steroids

[3 marks]

Identify TWO different diseases/disorders for which named drugs in use in the clinic
target enzymes which regulate the levels of endogenous steroids.
For EACH disease,

d) Describe the pathology associated with the disease/disorder and identify the
enzyme targetted;
The substrates and products of the enzymatic reactions;
How the drug acts at molecular, cellular, organ and organismal level to treat the
disease/disorder;

Disadvantages or common adverse effects of the use of the drug;
How the drug has been developed from predecessor versions to improve its
usefulness

[20 marks]

PHAR4007E1
2. Antibodies have become increasingly widespread as therapeutic alternatives to treat
diseases/disorders.

a) Briefly outline the properties of monoclonal antibodies that make them useful as
therapeutic options.

[2.5 marks]

b) Identify reservations or disadvantages about the use of monoclonal antibodies in
the clinic.

[2.5 marks]

c) Identify TWO antibodies designed to target named G proteincoupled receptors, the
tissue/cellular locations of those targets and the disease/disorder the antibodies are
used to treat

[2.5 marks]

d) Identify TWO antibodydrug conjugates and outline how they differ from simple’
monoclonal antibodies. Identify the molecular/cellular targets for their use, as well
as the disease/disorder they are intended to treat

[2.5 marks]

e) Discuss the treatment of cancers using antibodies and related biopharmaceuticals.
In your answer, compare the use of antibodies with small molecule drugs.
Making use of specific named examples,

Describe the pathology associated with the cancer, which is targetted by
treatment with those antibodies/drugs;
How the antibody/drug acts at molecular, cellular, organ and organismal level to
treat the cancer;
Contraindications, disadvantages or common adverse effects of the use of the
antibody/drug;
How the antibody/drug has been developed from predecessor versions to
improve its usefulness

[20 marks]

PHAR4007E1
End

SECTION B
(40 marks)

3. Calcium is an important regulator of cell function.
Outline the pathways through which calcium levels inside the cell can be regulated and
what impacts elevating intracellular calcium can have in different cell types.
Describe how named drugs can modify intracellular calcium levels for therapeutic gain
in specified diseases/disorders.

[20 marks]

4. Describe adaptations evoked by longterm exposure to receptor agonists and
antagonists in clinical use. In your answer, identify desirable and unwanted effects of
the named ligands used. Describe any mitigating approaches during therapy to account
for the adaptation phenomenon and strategies to deal with cessation of medication.

[20 marks]