Introduction

Question 1

Drug targets in treatment of human disease

Answer 1

Molecular triggers, ‘the architecture’ (enzymes, receptors, ion channels and transporters) since they impact intracellular signalling which influences physiological responses.

Specifically, druggable molecular targets are predominantly rhodopsin like GPCRs (~30%), Nuclear receptors, Ligand gated ion channels, and pathogens due to structural, chemical and pharmacological features.

Question 2

Drug discovery process //SLIDE 2?

Answer 2

Begins with basic research and drug discovery on which many thousands of higher targets or compounds are examined and developed. Each stage eliminates potential new medicines so there’s less and less. Need to understand what we are measuring, what this information gives us, and how this helps move to the next stage.

1. Target ID: Identify the targets that link to disease through genetics and known biology (ex. GPCR).
2. Target validation: Test lead compounds and targets in vitro. Assess affinity and selectivity through binding of a radioactive H ligand. Assess potency and efficacy through mode of action.
3. Lead discovery: Develop a therapeutic interacting with target to obtain disease cure/modification
4. Lead optimisation
5. Pre-clinical development
6. Clinical trials: Phase I in which there’s tens of volunteers, Phase II has hundreds and Phase III thousands
7. FDA review of typically the one final medicine that passed all stages.
8. Phase IV involves post-approval research and monitoring