Lecture 3

Question 0

Digitalis

Answer 0

Used to treat dropsy (congestive heart failure)

Question 1

Ma huang

Answer 1

Heart stimulant and for nasal and chest congestion

Question 2

Target identification

Answer 2

Identification of biological components that are potentially drugable

Question 3

Target validation

Answer 3

Determines whether interfering with the target is likely to have an impact on cancer growth

Question 4

Target to hit

Answer 4

Large numbers of chemical and biological agents are screened to identify molecules that hit the target

Question 5

Hit to lead

Answer 5

Hits are further tested to determine which bind the target with my specificity

Question 6

Lead optimization

Answer 6

Refining the properties of the lead molecules to enhance potency and reduce side effects

Question 7

Preclinical testing

Answer 7

Test for effectiveness of optimize lead

Question 8

Clinical trials

Answer 8

Determines whether it improves patient outcomes

Question 9

Phase 1

Answer 9

3 to 18 months

Evaluate safety, tolerability, PK and effects in healthy volunteers

Question 10

Phase 2

Answer 10

1-3 years.

Assesses effectiveness, side effects and other safety features and forms a dosing strategy and several HUNDRED patients

Question 11

Phase 3

Answer 11

2-6 years

Establishes efficacy and adverse effects from long-term uses and THOUSANDS of patients

Question 12

Phase 4

Answer 12

Continuous

Monitoring of effects contraindications and interactions reported by MD and Pharm.D.

Question 13

IND

Answer 13

Investigational new drug application

Before phase 1

Question 14

NDA

Answer 14

New drug application

After phase 3 before phase 4

Question 15

Lipitor

Answer 15

HMG CoA inhibitor

Cholesterol

Question 16

Plavix

Answer 16

Antiplatelet

Thrombosis

Question 17

Nexium

Answer 17

Proton pump inhibitor

Antiulcer

Question 18

Norvasc

Answer 18

Calcium channel blocker.

Hypertension

Question 19

What is out there and is there room for improvement?

Answer 19

Cure rather than alleviating symptoms the holy Grail.
Act on underlying pathophysiology. Greater efficacy.
New dosage form
Fewer side effects

Question 20

Ideal target

Answer 20

Disease modifying or proven function
Known 3-D structure
Easy assay allowing high throughput screening

Question 21

To be a target?

Answer 21

It has to be validated

Question 22

Gene knockout

Answer 22

Removal of the gene that encodes for the biomolecule of interest

Question 23

RNAi

Answer 23

Double-stranded RNA interfering with the expression of genes the sequences complementary to dsRNA

Question 24

Small Molecule Probe

Answer 24

Assay each compound against a disease model into you get a hit

Question 25

Animal models

Answer 25

Difficult, time-consuming, expensive

Question 26

Computation docking

Answer 26

Known, homologue or theorized structure of a protein can be modeled and molecules designed and tested for their interaction that may cause a confirmational change in the protein

Question 27

Proteomics

Answer 27

Provides a protein profile of the cell tissue or body fluid that can be used to compare

Question 28

Phenotype

Answer 28

Genotype
Natural variation
Environment

Question 29

Phenotypic screening

Answer 29

Link between genetic alteration and disease phenotype

IDs a cause rather than a consequence