Preclinical Models of Diseases for Drug Discovery

Question 1

What can animal models be used for?

Answer 1

• Establish PKPD relationships
• Evaluate MOA
• Discover new drug targets and biomarkers
• Estimate clinical dose regimes
• Determine safety margins and toxicity
• Characteristic disease pathophysiology
• Assessing the therapeutic utility of a drug

Question 2

How is the validity of a disease model validated?

Answer 2

Face validity - Shows similar symptoms to humans
Construct validity - Underlying biology of disease similar
Predictive validity - Animal model is responsive to clinically effective therapeutic agents

Question 3

What are the limitations of animal models?

Answer 3

• Dont model all aspects of human disease
• Difference in cellular mechanisms to humans
• Studies for safety and toxicity often done on one species / strain (not enough variaiblity)
• Animal model must be representative for purpose
• Must consider disease process in humans

Question 4

What are the types of epilepsy?

Answer 4

Idiopathic - Genetic
symptomatic - A result of damage
Provoked - caused by environment
Cryptogenic - unknown