Drug discovery

Question 1

Chemical drugs definition

Answer 1

Range from simple inorganic salts to complex organic molecules
Organic drug molecules are often referred to as small molecules, MW <500
New Chemical Entity

Question 2

Biological drugs definition

Answer 2

Large molecule drugs, usually proteins such as peptides or antibodies but could be a nucleic acid e.g. gene therapy
New Biological Entity

Question 3

Where do novel small molecules come from?

Answer 3

Natural sources or derived from nature
Derived from endogenous (biochemical) compounds
Synthetic drugs, derived from screening or rational medicinal chemistry
Serendipity and me-too drugs
In silico drug design- computer aided

Question 4

Plants are a rich source of medicinal natural products, some are active as isolated:

Answer 4

Paciltaxel- breast and ovarian cancer, isolated from bark of the yew tree
Quinine- malaria, isolated from bark of cinchona tree
Digoxin- heart failure and arrhythmias, leaves of the foxglove
Morphine- analgesic, contained within resin from poppy seeds

Question 5

Drugs may be derived from natural product lead compounds e.g. aspirin

Answer 5

Derived from willow bark, recorded use from 400BC to relieve fever, active ingredient in salicin
The lead compound has been developed into a clinically useful drug

Question 6

Problems with natural ligands

Answer 6

May have undesirable duration of action
May be excreted or metabolised too quickly
May be chemically unstable

Question 7

Sulfonamide group

Answer 7

SO2NH2

Question 8

Me-too drugs

Answer 8

A me-too drug has similar structure to an existing drug, modified enough to make the compound novel, but not so much that activity is affected

Question 9

Serendipity

Answer 9

The occurrence and development of events by chance in a happy or beneficial way

Question 10

Pharmacophore

Answer 10

The portion of a drug required for activity