Role of Academic labs

Question 1

What is a New Chemical Entity?

Answer 1

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.

Question 2

How long and how much does it cost to produce a new drug?

Answer 2

About 15 years and 1.5 billion dollars.

Question 3

What happens on Clinical Trial Phase 1?

Answer 3

The drugs is tested for safety and pharmacokinetics in volunteer humans.

Question 4

What happens on Clinical Trial Phase 2?

Answer 4

The drug is tested on larger groups of humans for dosage requirements and pharmacodynamics.

Question 5

What happens on Clinical Trial Phase 3?

Answer 5

The drug is tested with large groups of humans in order to establish if the drug is better than the gold standard?treatment.

Question 6

What is the Proof of Concept performed on phase 2 of clinical trials?

Answer 6

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.

Question 7

What is the challenge presented with NCE?

Answer 7

The challenge is to find a drug that is worth the investment, without making large investments in targets and drugs that will ultimately fail.

Question 8

What is an example of an academic discovery?

Answer 8

3-hydroxy-3-methyl-glutaryl-CoA reductase in the metabolism of cholesterol lead to the production of statins.

Question 9

What are statins?

Answer 9

Mevacor, Lovastatin and Zacor by Merck block the receptor for the resucitase and reduce colesterol in the blood.

Question 10

What is confidence in rational and safety?

Answer 10

It presents evidence that a certain drug hits the target in an effective and safe way.

Question 11

Why statins needed confidence in rational and safety?

Answer 11

Because mice developed tumours in the liver.

Question 12

What is the most famous medicine in the world?

Answer 12

Atorvastatin

Question 13

What is an example of another NCE but statins?

Answer 13

Rutixan. It was used to treat multiple sclerosis and now it is used to treat cancer.

Question 14

What are three technologies developed in academic labs:

Answer 14

siRNA-used in the validation of targets
Embryonic stem cell-in vitro models of diseases
CRISPR- used to gene editing

Question 15

What large companies can do that labs can’t?

Answer 15

Excel the later stages of processes in product developments such as clinical trials.

Question 16

Why do some drugs actually fail in development?

Answer 16

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

Question 17

What is an example of a withdrawal?

Answer 17

Pfizer Torcetrapib to treat cholesterol.

Question 18

Which universities are showing progress as academic labs per clinical trials?

Answer 18

Ohio State
Mount Sinai
Duke University
San Francisco UC

Question 19

What are two recommendations for academic labs?

Answer 19

1. To concentrate in basic research that may lead to innovative discoveries and targets.
2. Develop a good confidence of rationale and safety.

Question 20

What are the characteristics of a good NCE?

Answer 20

Solubility
Cellular compatibility
Pharmacokinetics
Adequate dosage
Low toxicity and proof of concept

Question 21

What is a new chemical entity?

Answer 21

It is anne drug that is considered safe, effective, and has been approved or also an approved drug that has a new use.

Question 22

What is a copycat drug?

Answer 22

It is a drug that acts over the same target with the same MOA.

Question 23

What is Standard Activity Relationship?

Answer 23

It is the production of multiple entities of similar chemical constitution and are tested for effectiveness.

Question 24

What is a hit?

Answer 24

A chemical entity that produces the desired effect.

Question 25

What is a lead?

Answer 25

It is the chemical scaffold of a molecule where functional groups are altered and manipulated to improve the drug’s functioning.