5. Preclinical Studies in Drug Development

Question 1

Legal requirement for preclinical studies

Answer 1

Requires that non-human animal research occur to show the safety and efficacy of new treatments before any human research will be allowed to be conducted

Question 2

3 R’s principles in animal research

Answer 2

Replacement

Reduction

Refinement

Question 3

Translational Medicine

Answer 3

A category of biomedical research that aims to improve human health

• Identify & invent diagnostic tests
• Drug & therapeutic development
• Clinical impact
• interdisciplinary

Question 4

Xenograft

Answer 4

a tissue graft or organ transplant from a donor of a different species from the recipient

Question 5

M4K

Answer 5

Medicine for Kids Pharma (Open science)

Question 6

How are xenograft mouse models used for testing cancer drugs?

Answer 6

healthy mice are injected with human cancer cells
& mice are then treated with a drug(s) to shrink tumors

Question 7

How many novel compounds were found against mutant ACVR1 receptor

Answer 7

Found 2 chemicals that were good at selectively blocking signals set out be ACVR1 and killing ACVR1-mutant cells
(extended survival by 25 per cent )

Question 8

ALS (Amyotrophic lateral sclerosis)

Answer 8

Progressive & fatal disease that causes motor neurons to break down

Question 9

What model animal was used to find a treatment for ALS

Answer 9

Worms have been genetically engineered to mimic one type of genetic cause of human motor neuron disease (ALS)

Question 10

Model animals in Preclinical Studies for new drugs to treat anxiety

Answer 10

Mice prefer dark and enclosed spaces (nocturnal species)

Question 11

ACVR1 signaling pathway in DIPG

Answer 11

• Is a cell surface receptor
  
  • Responds to ligand
  • Cascade turns on downstream genes
    In DIPG there is a mutation in the kinase domain of ACVR1, creating a mutant ACVR1 protein with different signaling properties

Question 12

DIPG

Answer 12

a rare brain cancer