Role of Academic labs Flashcards
What is a New Chemical Entity?
It is a drug that is proven to be safe, effective, and has been approved, or an already approved drug that has a different new use.
How long and how much does it cost to produce a new drug?
About 15 years and 1.5 billion dollars.
What happens on Clinical Trial Phase 1?
The drugs is tested for safety and pharmacokinetics in volunteer humans.
What happens on Clinical Trial Phase 2?
The drug is tested on larger groups of humans for dosage requirements and pharmacodynamics.
What happens on Clinical Trial Phase 3?
The drug is tested with large groups of humans in order to establish if the drug is better than the gold standard?treatment.
What is the Proof of Concept performed on phase 2 of clinical trials?
It is the phase where the drug is demonstrated to have a module leads to a desirable clinical outcome in terms of efficacy, safety and dosage.
What is the challenge presented with NCE?
The challenge is to find a drug that is worth the investment, without making large investments in targets and drugs that will ultimately fail.
What is an example of an academic discovery?
3-hydroxy-3-methyl-glutaryl-CoA reductase in the metabolism of cholesterol lead to the production of statins.
What are statins?
Mevacor, Lovastatin and Zacor by Merck block the receptor for the resucitase and reduce colesterol in the blood.
What is confidence in rational and safety?
It presents evidence that a certain drug hits the target in an effective and safe way.
Why statins needed confidence in rational and safety?
Because mice developed tumours in the liver.
What is the most famous medicine in the world?
Atorvastatin
What is an example of another NCE but statins?
Rutixan. It was used to treat multiple sclerosis and now it is used to treat cancer.
What are three technologies developed in academic labs:
siRNA-used in the validation of targets
Embryonic stem cell-in vitro models of diseases
CRISPR- used to gene editing
What large companies can do that labs can’t?
Excel the later stages of processes in product developments such as clinical trials.
Why do some drugs actually fail in development?
They fail due to toxicity issues in animals in the early stages.
70% fail at phase 2
50% fail at phase 3
They are inadequate and have safety issues
What is an example of a withdrawal?
Pfizer Torcetrapib to treat cholesterol.
Which universities are showing progress as academic labs per clinical trials?
Ohio State
Mount Sinai
Duke University
San Francisco UC
What are two recommendations for academic labs?
- To concentrate in basic research that may lead to innovative discoveries and targets.
- Develop a good confidence of rationale and safety.
What are the characteristics of a good NCE?
Solubility Cellular compatibility Pharmacokinetics Adequate dosage Low toxicity and proof of concept
What is a new chemical entity?
It is anne drug that is considered safe, effective, and has been approved or also an approved drug that has a new use.
What is a copycat drug?
It is a drug that acts over the same target with the same MOA.
What is Standard Activity Relationship?
It is the production of multiple entities of similar chemical constitution and are tested for effectiveness.
What is a hit?
A chemical entity that produces the desired effect.
What is a lead?
It is the chemical scaffold of a molecule where functional groups are altered and manipulated to improve the drug’s functioning.
