Introduction Flashcards
Describe 2 scientific approaches
Hypothesis driven - “I think x is caused by y, so let’s test it”
Exploratory - “I was looking to see if x is caused by y but noticed z”
Describe the multi-factorial nature of research?
Outline why academia and industry seek to identify new therapeutic targets?
- New drug types – small molecules vs biologics
- New ways of targeting signalling, inc. anti-peptide antibody
- New formulations/delivery methods
- New drug combinations
- New applications therefore new conditions to treat – diseases/patient groups
- New markers for selection of patients that would respond better to therapy being tested/diagnostics (Personalized medicine)
- New understanding of biological functions – as old drugs usually have multiple targets
What are the ultimate goals of a therapeutic agent?
- To meet the unmet medical needs
- To be more efficacious
- To have fewer side effects
- To production more profit
Demonstrate an example of competition in the pharmaceutical industry?
Company x produces a drug, company z plays around w/side chains to produce a more efficacious drug than the original
How can we develop new therapeutic targets?
With knowledge of disease mechanisms (+ genomics + protein structures) which allow us to select targets and search for drug leads and candidates
Explore targets of (un)successful drugs since existing targets help:
Molecular dissection of action mechanisms
Prediction of features that guide new drug design
Advances in new technologies/tools for these tasks
Why is a therapeutic target with relevant signalling difficult to come by?
Give an example.
(Part 1)
Because it still needs:
1) Effective and selective targeting
2) Clinical impact
3) Good benefit: risk ratio (=not much side effects)
4) Needs to be scaled up for mass production
Example: Rimonabant is an anti-obesity drug
Rimonabant is an inverse agonist for the cannabinoid receptor CB1 and was the first drug approved in that class.
Rimonabant is an inverse agonist for the cannabinoid receptor CB1 and was the first drug approved in that class.
Why was it removed?
(Part 2)
It has been removed from the market because although it is reasonably effective it has been seen to cause severe clinical depression and suicide ideation
Describe the process of drug development
Does the process of drug development end at phase 3?
No, there is Post legislation pharmaco-surveillance (help identify additional drug-drug interactions and potential side effects) -> Phase IV
What is the use of clinical trial databases?
https://clinicaltrials.gov/ - those would tell you about any ongoing, completed or just started recruitment
http://pubchem.ncbi.nlm.nih.gov/
You search them for targets or disease states or drug type – for example type MS to find about trials re: MS
Describe how databases can be used to facilitate drug discovery and development?
- To find interventional Phase 3 clinical trials for a condition like migraine
- To identify types of therapeutic agents currently undergoing clinical trials
- To see if something has good translation potential?
How are drugs named? Give an example
When a drug is approved by the FDA it is given a:
(a) Generic (official) name e.g.: Ibuprofen
(b) Brand (proprietary or trademark or trade) name e.g.: Brufen or advil
Who funds research?
Charities, research council, each agency will be willing to fund a certain area of research
Top NIH-funded disease areas?
Explain the funding available for drug discovery and development
Cancer, Infectious diseases, Brain disorders, Rare diseases
Sometimes individuals fund research regarding a certain disease for example Huntington disease was running in a rich family so they funded its research and therefore there is more clinical information on it
