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S1 Pharmacology > Drug Targets > Flashcards

Drug Targets Flashcards

1
Q

“EMPIRICAL’ Drug-Discovery

A
  • Recognize initial drug lead by functionally useful effect
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2
Q

Examples of empirical drug discovery

A
  • Penicillin (anti-bacterial effect);
  • Taxol (anti-tumor)
  • Digoxin (cardiotonic / antiarrythmic)
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3
Q

“RATIONAL Drug discovery

A
  • Drug by design or screen against biochemical target’s function
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4
Q

Examples of rational drug discovery

A
  • HIV-protease inhibitor (anti-infection)
  • Metoprolol (anti-hypertensive)
  • Methotrexate (anti-tumour)
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5
Q

Major causes of Drug Failure in Phase II

A

• Wrong target?
• Wrong patient population?
• Wrong dose?

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6
Q

What is a Drug Target?

A

A bio-molecule which either:
– Is present in the diseased tissue
– Has elevated expression in the diseased tissue
– Is overactive in diseased tissue
– Has a function contributing to development or existence of disease
– Has an involvement or role in disease process

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7
Q

Drug Targets and Mechanisms

A
  • Enzymes Reversible = Irreversible Inhibitors
    • Receptors = Agonists & Antagonists
    • Viral Surface Proteins = Block entry into cell
    • Ion channels = Block or Open channel
    • Transporters = Block or promote transport
    • Nucleic Acids = Inhibit function, prevent gene expression
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8
Q

Targets for Drug Action

A
  • look at slide 10
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9
Q

What factors must pharmaceutical companies consider when developing drugs?

A

• Both economic and medical factors.
• They must ensure a financial return on investment.

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10
Q

What caused a rise in antimalarial research by pharmaceutical companies?

A

• Growth in tourism to exotic countries.
• The spread of malaria to regions affecting wealthier populations.

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11
Q

Why do pharmaceutical companies focus on diseases with few treatment options?

A

• To address unmet medical needs.
• To create drugs with better properties than existing ones.
• Ensures competitive advantage in the market.

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12
Q

What makes a good target?

A
  • Must be differentially expressed / regulated/ located in diseased tissue
    • Must be central to disease process with robust studies in clinical samples
    • Must be characterised in terms of expression, activity, function and role
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13
Q

Why does disease-association not make a protein a viable drug target?

A
  • The protein must be validated as a point of intervention in the disease pathway
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14
Q

What roles do bioinformatics and cheminformatics play in drug discovery?

A

• Bioinformatics: Identifies and analyzes disease-related targets using biological data.
• Cheminformatics: Helps design and optimize drug-like molecules by analyzing chemical data.

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15
Q

Target Validation Process

A
  • Disease
  • Target
  • Target Selection
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16
Q

Drug Discovery Process

A
  • Target Selection
  • Leads
  • Clinic
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17
Q

Target Validation

A
  • Identifies and assesses whether a molecular target merits the development of drugs
  • process of demonstrating that a molecular target is a therapeutically relevant pharmacological target
18
Q

What is the most important part of drug discovery ?

A
  • Target validation
19
Q

A Valid Target is:

A
  • A target that when modulated pharmacologically, provides meaningful efficacy and acceptable safety for specific human disease in long-term clinical usage
20
Q

Target Validation is:

A
  • The process of demonstrating that engaging the target provides statistically meaningful therapeutic benefit with acceptable safety for a given indication.
21
Q

Proof of Concept is:

A
  • Preclinical or limited clinical studies prior to well-powered clinical trials, that establish the scientific validity and safety of a drug target; it is part of the
    continuum of target validation.
22
Q

Target Identification is:

A
  • The generation of scientific evidence that a manipulatable able target is involved in some significant way in a disease process
23
Q

What is the purpose of target validation?

A
  • Increases our confidence in the relationship between the target and the disease.
  • Demonstrates target is critical or central to disease development or progression.
  • Allows exploration of effects caused by modulation of target, to identify mechanism based adverse effects
24
Q

Target validation of human data

A
  • clinical experience (most important)
  • genetics
  • tissue expression
25
Q

Target qualification of preclinical data

A
  • translational endpoints (most important)
  • genetically engineered models
  • pharmacology
26
Q

Target expression relationship

A

Slide 20

27
Q

Genetic relationship to disease

A

Slide 21

28
Q

Clinical trial data and validation

A

Slide 22

29
Q

Preclinical pharmacological validation

A

Slide 23

30
Q

Preclinical genetic validation

A

Slide 24

31
Q

Clinical translatability of preclinical data

A

Side 25

32
Q

How do you identify the target?

A
  • Genomics
  • proteonomics
  • gene association
33
Q

How can genetics help identify a valid target?

A

• the study of SNPs (single nucleotide polymorphisms) and polymorphisms that link genetic variations to disease.

34
Q

How are in vitro models used to validate a target?

A
  • Techniques like siRNA
35
Q

How are in vitro models used to validate a target?

A
  • Techniques like siRNA
36
Q

What role does clinical data play in target validation?

A
  • disease database
37
Q

How do expression patterns contribute to target validation?

A
  • Location, Disease modulation, Microarray studies
38
Q

How are in vivo models used to validate a target?

A
  • Transgenic, Null / Knock-out mice, Behavioural models
39
Q

What is the role of model organisms in target validation?

A
  • Organisms like Drosophila, zebrafish, and C. elegans are used to study the biological roles of targets in simpler systems.
40
Q

How do ‘omics’ technologies assist in target identification?

A
  • fields like genomics, proteomics, and transcriptomics provide large-scale data on gene, protein, and transcript interactions with disease
41
Q

Identifying the actual drug target

A
  • detection of ‘new’ drug target in samples of diseased tissue
  • compare expression of ‘new drug targets in many clinical samples
  • creation of a test model

S1 Pharmacology (33 decks)

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