Therapeutic Concepts

Question 1

What are the key drivers of drug discovery and development?

Answer 1

• Scientific curiosity and advancement
• Need for new treatments
• Profitability
• Socioeconomic aspects

Question 2

Importance of 3D structures in drug design

Answer 2

• Information about the molecular basis of disease
• Binding sites
• Protein interactions
• Target flexibility
• Ligand binding and unbinding kinetics

Question 3

Needs and opportunities for new therapeutic agents

Answer 3

• Advances in biotechnology
• Existing drugs may have side effects/toxicity
• Need for personalised medicine due to genetics/lifestyle factors
• Investments in R&D drive innovation
• Greater understanding of disease mechanisms
• Drug resistance
• Improved screening methods

Question 4

Therapeutic effect (definition)

Answer 4

Therapeutic effect is the intended, positive impact a drug has on the body, it is an endpoint clinicians want to achieve by prescribing a particular medication.

Question 5

Concepts influencing therapeutic efficacy

Answer 5

• Efficacy: Tested in clinical trials
• Selectivity: Selective drugs are typically more effective and less toxic than non-selective drugs
• Pharmacokinetics (ADME): How the drug is absorbed, distributed, metabolised, and excreted by the body
• Pharmacodynamics: The molecular, biochemical, and physiological effects of the drug + mechanism of action
• Toxicity: Acute toxicity can be life-threatening, while chronic toxicity can lead to serious health problems, such as organ damage

Question 6

Hit and lead compound identification

Answer 6

• High-throughput screening
• Natural products
• Computational virtual screening
• Structure-based DD
• De novo DD
• Fragment-based approaches
• In silico screening