Medicinal Chemistry

essentially descending order of importance, these drugs and druggable targets may be
classified as follows:

1. Proteins, peptides, amino acids
   Enzyme proteins
   a. As drugs
   Alteplase (for ischemic stroke)
   Retavase, tenecteplase (for acute myocardial infarction)
   b. As druggable targets
   Classical enzyme targets (acetylcholinesterase, monoamine oxidase)
   Emerging targets (kinases, caspases)
   Non-enzyme proteins
   a. As drugs
   Insulin, growth hormone
   b. As druggable targets
   Protein folding disorders (β-amyloid, prions)


2. Nucleic acids, nucleotides, nucleosides
   a. As drugs
   Antimetabolites
   Antisense oligonucleotides
   b. As druggable targets
   Traditional antineoplastic/antiviral agents targeting nuclear DNA
   Agents targeting ribozymes and RNA targets


3. Lipids
   a. As drugs
   Steroids (estrogens, progestins, androgens, corticosteroids)
   Terpenes (pheromones)
   b. As druggable targets
   Anti-prostaglandin agents (cyclooxygenase inhibitors)
   Anti-leukotriene agents (5-lipoxygenase inhibitors)

482 MEDICINAL CHEMISTRY

Figure 8.5 Macromolecular targets for drug design. The mammalian cell contains numerous
macromolecules including lipids, enzymatic proteins, non-enzymatic proteins, carbohydrates,
nucleic acids, other heterocycles, and water. All of these molecules present important opportunities
for drug design.