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Medicinal Chemistry > Topic One-Intro And Drug Discovery > Flashcards

Topic One-Intro And Drug Discovery Flashcards

1
Q

What is a drug

A

Compounds that interact with a biological system to produce a biological response

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2
Q

What is drug selective toxicity

A

Toxic to problem cells not to normal cells

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3
Q

What are the 4 groups of drugs

A

Natural products
Fermentation products
Semi synthetic- produced by partial synthesis
Completely synthetic

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4
Q

Modern drug discovery

A

Advances in technology, combinatorial Chemistry, and molecular biology paved the way. For high-throughput screening and rational drug design. Target based drug discovery and the use of computational methods became integral to the process

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5
Q

Drug target

A

As life is made up of cells drugs must act on cells
The main molecular targets for drugs are proteins (mainly enzymes, receptors, and transport proteins) and nucleic acids (DNA & RNA)

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6
Q

Typical molecular weight of a drug

A

Less than 500 atomic mass units

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7
Q

What are drug targets at the molecular level

A

Large macromolecules (usually proteins) with molecular weights in the thousands of atomic mass units

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8
Q

What is a binding site

A

A hollow region on the macromolecular target where the drug fits and binds

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9
Q

What type of bond is the strongest in drug- target interaction

A

Covenant, strength of 200-300 KJ/Mol

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10
Q

5 types of intermolecular bonds involved in drug binding

A

Ionic bonds (20-40 KJ/Mol)
hydrogen Bonds (16-60 KJ/mol)
London bonds (2-4 KJ/Mol)
Dipole-dipole
Hydrophobic interaction (0.1-0.2 KJ/Mol)

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11
Q

What is induced fit in drug targets interaction

A

When a target adjusts its shape slightly to accommodate the drug during binding

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12
Q

What does the binding of a drug to its target result in

A

Formation of a bound complex through intermolecular interactions which can initiate a biological effect

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13
Q

Are covalent bonds reversible

A

Usually not

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14
Q

Are intermolecular bonds reversible

A

They are weaker so reversible and more common

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15
Q

What is the goal of drug discovery

A

To identify a specific biological target involved in a disease and find initial compounds that interact with it

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16
Q

What is a hot compound in drug discovery

A

An initial compound that shows interaction with the chosen biological target

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17
Q

What does QSAR stand for

A

Quantitative Structure-Activity Relationships

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18
Q

What are the main goals of drug design

A

Improve efficacy safety and pharmacokinetics
Evaluate safety and efficacy in preclinical studies

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19
Q

What happens in phase 1 of drug design

A

Evaluate safety dosage side effects in a small group of healthy volunteers

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20
Q

What is tested in phase 2 of clinical trials

A

The drugs Effectiveness and side effects in a larger group of patients with the disease

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21
Q

What’s the focus of phase 3 in clinical trials

A

Comfim drugs efficacy monitor side effects and compare it with standard treatments in a large population

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22
Q

What is a NDA

A

New drug application - a request to regulation agencies to approve the drug based on trial data

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23
Q

What’s the purpose of post-marketing surveillance

A

To monitor the drugs safety and effectiveness in real-world settings after it’s on the market

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24
Q

What is target identification

A

The process of identifying specific biological molecules associated with a disease and validating their relevance for drug development

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25
What is disease understanding
Understanding the molecular and cellular basis of a disease
26
Genomic approaches
Genomic data analysis, including GWAS (Genome-Wide Association Studies) can help identify potential genetic factors linked to the disease
27
Proteomics approaches
The use of proteomic techniques can identify proteins that play a role in the disease
28
Proteomics
Study of all the proteins in a cell, tissue or organism
29
Genomics
Study of the entire genome of an organism Involves- Mapping location of genes Sequencing DNA analyzing gene functions and interactions Understanding how genes contribute to health disease and individual traits
30
Pathway analysis
Can identify key signalling pathways associated with the disease
31
Target validation
Explaining the process of validation the severed target to confirm its role in the disease
32
Genetic validation
Genetic methods like knockout studies can confirm the impact of the target in the disease phenotype
33
Pharmacological validation
Pharmacological tools such as small molecules or antibodies can be used to modulate the targets activity
34
Hit generation
Potential compounds, called hits, are identified as having the potential to interact with a specific biological target
35
Methods for hit generation
High-throughput screening (HTS) Virtual screening Fragment-based screening
36
High-Throughput Screening (HTS)
The use of automated equipment to rapidly test thousands to millions of samples for biological activity at the model organism, cellular, pathway, or molecular level
37
Virtual screening
Virtual screening as a computational approach that uses computer models to predict how well certain compounds may bind to a target
38
Fragment based screening
Smaller chemical fragments are screened for binding, and hits are built up from these fragments
39
Hit-to-lead Optimisationt
Initial hits are further refined to develop lead compounds with improved potency selectively and pharmacokinetic properties Efficacy- improved potency and biological activity Selectively- reduced off target effects ADME Properties- enhanced pharmacokinetic characteristics Safety- initial assessment of toxicity
40
Lead optimisation
Selected lead compounds undergo further refinement to develop a drug candidate with optimal therapeutic properties
41
Medicinal chemistry
Continued refinement of chemical structures based on structure activity relationship (SAR) studies
42
Toxicology studies
Assessing and addressing the potential safety issues
43
Bioavailability
Enhancing the drugs ability to reach the target in the body
44
Metabolic stability
Ensuring the compound remains intact in the body for an appropriate duration
45
Half life
Extending the time the drug stays in the blood stream
46
Dose-Response relationship
Understanding how the drugs effect can change with different doses
47
Preclinical development
Selected drug candidates undergo extensive testing in non-human systems to assess safety, efficacy, and pharmacokinetics before advancing to human clinical trials
48
Key points of preclinical development
Evaluate safety and toxicity Assess pharmacokinetics and pharmacodynamics Establish safe starting dose of human trials Gather data for regulatory submissions
49
In vitro studies
Testing in isolated cell cultures to understand mechanisms
50
In vivo studies
Testing in living organisms to assess overall pharmacological effects
51
Key points of phase 1 clinical trials
1) objective- assess safety, dosage, and pharmacokinetics in a small group of healthy volunteers 2) ppts- usually 20-100 healthy volunteers 3) duration- short duration (several weeks) 4) endpoint- focus on safety, dosage range and initial pharmacokinetic data 5) regulatory milestone- submission of a Investigstional New Drug (IND) application
52
Key points of phase 2 of a clinical trial
1) objective- assess efficacy and further evaluate safety in a larger group of patients with the target condition 2) ppts- a few hundred patients 3) duration- several months to a year 4) endpoint- assess efficacy, optimal dosage, and continue safety analysis 5) regulatory milestone- if successful, may lead to conditional approval for continued testing
53
Key points for phase 3 of a clinical trial
1) objective- confirm efficsy, monitor adverse reactions snd further assess safety in a larger more diverse ppt population 2) ppt- involves hundreds to thousands of patients 3) duration- can span several years 4) endpoint- robust assessment of efficacy, safety, and identification of potential rare side effects 5 regulatory milestone- submission of NDA for regulatory approval
54
Weaknesses of drug design
Challenging Expensive Time consuming
55
How to lessen the weaknesses of drug design
Multidisciplinary approach
56
Multidisciplinary approach
Computational tools, methodology for structure guided approach & global gene expression data analysis by software
57
Strengths of multidisciplinary approach
Efficiency increased Cost effective Less time consuming Strategies to overcome toxic side effects
58
Bioinformatics
Is an interdisciplinary field that combines biology, computer science and information technology to analyse and interpret biological data, it plays a key role on target identification and computer screening of chemical compounds
59
2 ways of drug designing
1) development of ligands with desired properties for targets having known structure and functions 2) development of ligand with predefined properties for targets whose structural info may or may not be known
60
Databases
PDB- experimentally determined structures NCI- National Cancer Institute Pubchem- Bulk data available for QSAR for lead discovery OSIRIS- to draw chemical structures and predict drug like properties like absorption in the body
61
Key points on discovery and development
The time from conception to approval of a new drug is typically 10-15 years The vast majority of molecules fail The estimated cost to bring a new drug to market is 800 million

Medicinal Chemistry (1 decks)

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