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a. SL12101: The chemistry of drugs > L30 - New & Emerging Drug Technologies > Flashcards

L30 - New & Emerging Drug Technologies Flashcards

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1
Q

What does a mutation in DNA result in?

A

Defective protein requires for normal cell function
= causes disease

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2
Q

What are some diseases are exacerbated by?

A

Increased levels of particular proteins

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3
Q

What can sub-physiological levels of protein be overcome by?

A

Delivering exogenous protein

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4
Q

What can nucleic acids be used as?

A

Treatment in all scenearios

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5
Q

What does therapeutic protein overcome?

A

Difficulties of producing & purifying recombinant proteins
- getting patient’s cells to do it
- transicent - repeated injections of gene/vector requires

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6
Q

What is siRNA? What does it do?

A

Small interfering NRA
- knocks down gene expression if sequence is known

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7
Q

What are advantages of siRNA?

A
  • readily designed
  • readily synthesised
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8
Q

What is miRNA? What is it derived from?

A

Micro RNA
- genes that specifically code miRNAs (not proteins

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9
Q

What are miRNA important in?

A

Regulating gene expression (1/3 of genes)

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10
Q

What are advantages of miRNA?

A
  • dont cause mRNA destructions (repress or destabilise)
  • strong evidence of involvement in diseases states (inc. cancer)
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11
Q

What can be delivered to cells to replace missing miRNAs?

A

DNA coding underexpressed or missing miRNAs

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12
Q

What can overexpressed miRNAs be inhibited by?

A

Anti-miRNAs
- complementary nucleotides that bind to the problem miRNA

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13
Q

What are RNA vaccines?

A
  • viral mRNA absorbed by cells
  • expressed on surface
  • labelled as foreign cells
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14
Q

What is cell therapy?

A

Delivery of cells to the patient to correct disease or damge
- may be combined with genetic strategies (secrete therapeutic protein, reprogramming immune cells)

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15
Q

What is strimvelis? What does it treat?

A

First ex vivo stem cell gene therapy
- treats severe combined immunodeficiency cause by adenosine deaminase deficiency
- autologoes CD34+ cells expressing functional ADA
- (rare)

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16
Q

How are cell therapies with CAR T cells done?

A
  • remove blood from patient (get T cells)
  • make CAR T cells in lab
  • grow millions
  • infuse CAR T cells into patient
  • CAR T cells bind to cancer cells, killing them
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17
Q

What are approved CAR T cell therapies?

A
  • kymriah
  • yescarta
  • tecartus
  • breyanzi
  • ABECMA
  • carvykti
18
Q

What are emerging cell therapies?

A
  • TCR-T cells
  • CAR-M
  • CAR-NK cells
19
Q

How do CAR T cells work?

A
  • recognise antigen on tumour cell
  • causes transcription of T cell effector function
    = perforin and granzyme release
20
Q

What was combination cell-gene therapy?

A
  • trialed in 56 patients
  • mesenchymal stem cells transfected with gene (TRAIL)
  • off the shelf treatment
  • scaling up cell culture (key)
  • funded through the biomedical catalyst (MRC and Innovate UK)
21
Q

What are current approaches to nucleic acid therapy like?

A

Often transient
- varying degrees of success

22
Q

What is gene editing?

A

Permanently editing the genome to correct a defect, knocking down a specific gene/activating a gene

23
Q

What are ways of gene editing?

A

Break in double strand break
- gene disruption
- gene correction
- gene insertion

24
Q

What is an example of gene editing? What does it do?

A

CRISPR/Cas9 (system from bacteria can be used to edit DNA)
- modifies genes in human cells in vitro and in vivo
- used to inactivate, correct and insert genes

25
Q

How could gene editing cure HIV?

A

Cause mutation in CCR5 expression
- prevents HIV from entering cells as not expressed on T cells

26
Q

What are examples of stem cell therapies?

A
  • totipotent
  • pluripotent
  • multipotent
27
Q

What is stem cell therapy to cure blindness?

A

Limbal stem cell transplantation
- cure blindness due to corneal burning

28
Q

What does tissue engineering aim to do?

A

replace diseased or damaged livgin tissue with living tissue designed and contructed for the needs of each individual

29
Q

What are the steps in tissue engineering?

A
  • isolate cells
  • expand cell number
  • seed on suitable scaffold
  • culture under suitable conditions to generate a mature tissue
  • implant tissue in patient
30
Q

What is a way of tissue engineering for implants?

A

3D bioprinting

31
Q

What is controlled drug delivery used for?

A
  • long term treatment (entrap drug in polymer matrix, dif shapes and szes)
  • small quantites released over a long period
32
Q

What are way of controlled drug delivery?

A
  • polymer degrades over time, releasing drugs
  • polymer doesnt degrade, drug slowly diffues
    (Microchips)
33
Q

Why would controlled drug delivery be favoured?

A
  • sustained therapeutic plasma conc
  • improved compliance - patient can’t forget dose
34
Q

What does targeted drug delivery aim to do?

A
  • accumulate and retain drug at site of action
  • minimise loss in transit
  • protect drug from premature clearance
  • aid intracellular delivery (in necessary)
35
Q

What are antibody-drug conjugates (ADCs)?

A

mAb therapy
- need to be combined with small drug chemotherapy/radiotherapy
- binds to specific targets, can deliver highly toxic drugs to site of action

36
Q

What are ADCs being developed for?

A

Delivery of chemotherapeutics to cancerous cells

37
Q

What are the current ADCs like on the market?

A

2 on the market, many nearing approval
- multiple drug molecules attaches via cleavable linker

38
Q

What is the Kadcyla ADC?

A

Combination os trastuzamab and cytotoxic drug
- increase median overall survival in HER2+ metastatic breast cancer by almost 6 months

39
Q

What are oncolytic viruses?

A

Viruses that break apart cancerous cells
- infect cancerous cells and cause lysis and apoptosis

40
Q

What does the enhanced permeability and retention have an effect on (EPR)?

A
  • blood vessels in tumours are leaky
  • macromolecules and nanoparticles easily enter, poorly drained
  • accumulation in tumours is possible
41
Q

How could you use bacteria to fight cancer?

A
  • oxygen deficient (hypoxic) areas, resistant to chemo/radiotherapy
  • some are olbigate anaerobes, can colonise hypoxic areas
42
Q

What is an examples of a bacteria that could fight cancer?

A

Probiotic-guided CAR (ProCAR)-T cells

a. SL12101: The chemistry of drugs (27 decks)

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