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Test 1 Flashcards

1
Q

What is a first-in-class drug

A

a drug that targets a protein that hasn’t been targeted by another drug

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2
Q

Are me too drugs or first in-class drugs more common?

A

me too

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2
Q

What is a me-too drug

A

a drug that targets a protein that has been targeted by another drug

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2
Q

What are the drug development pipeline steps?

A
  1. Discovery/ Preclinical Testing
  2. File IND at FDA (to get approved for humans)
  3. Phase 1
  4. Phase 2
  5. phase 3
  6. File NDA at FDA
  7. FDA
  8. Phase 4
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2
Q

How many years is discovery/ preclinical testing?

A

6.5

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3
Q

What is the test population in the discovery/ preclinical testing?

A

lab and animal studies

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4
Q

What is the purpose of discovery/ preclinical testing?

A

asses, safety, biological activity, and formulation

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5
Q

What is the success rate of discovery/ preclinical testing?

A

5,000 compounds evaluated

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6
Q

What is IND

A

investigational new drug

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7
Q

How many years is Phase 1

A

1.5

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8
Q

What is the test population of Phase 1?

A

20 to 100 healthy volunteers

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9
Q

What is the purpose of Phase 1?

A

determine safety and dosage

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10
Q

What is the success rate of Phases 1, 2, and 3?

A

5 out of 5,000 compounds

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11
Q

How many years is Phase 2?

A

2

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12
Q

What is the test population of Phase 2?

A

100 to 500 patient volunteers (have the disease)

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13
Q

What is the purpose of phase 2?

A

evaluate effectiveness and look for side effects

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14
Q

How many years is Phase 3?

A

3.5

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15
Q

What is the test population of Phase 3?

A

1000 to 5000 patient volunteers

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16
Q

What is the purpose of Phase 3?

A

confirm effectiveness, monitor adverse reactions from long-term use

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17
Q

What is an NDA

A

New Drug Application

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18
Q

How many years is the drug in the FDA?

A

1.5

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19
Q

What is the purpose of the drug pipeline?

A

reduce risk

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20
Q

What are the main therapeutic areas where drugs are being approved?

A
  1. Oncology
  2. infectious disease
  3. neurology
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21
Q

What is the List of Essential Medicines?

A

world health organization

a list of medicines that satisfies the priority health care needs of the population

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22
Q

What does the World Health Organization take into account?

A
  • how common the disease is
  • how serious the disease is
  • evidence that the drug works
  • evidence the drug is safe
  • cost-effective drugs (affordable)
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23
Q

How do patients help shape the pipeline?

A
  • doctors have focus groups and ask patients which side effect is the worst with their disease
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24
Q

Why does drug development have immense attribution?

A

Start with 5,000 compounds and narrow down to only one

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25
Q

What are some considerations in early drug development?

A
  • medical need (seriousness of disease)
  • availability of current therapy
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26
Q

What is program selection?

A

choose a disease

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27
Q

What is a target drug?

A

a molecule (usually protein) involved in the disease, that the drug binds to, often inactivation the molecule. (a molecule we aim our drug at)

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28
Q

What happens after a target drug is identified?

A

test many thousands of chemicals against it

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29
Q

What is the design of Phase 1 trials?

A

slowly increasing dose

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30
Q

What is the design of Phase 3 trials?

A

compare treated individuals to control individuals (with the use of placebos)

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31
Q

Why do Phase 2 trials to blind experiments?

A

reduce bias

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32
Q

What is single-blind?

A

subject don’t know who’s getting placebo

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33
Q

What is double-blind?

A

Neither subjects or doctors know who got the placebos

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34
Q

What is the design of Phase 3?

A

usually randomized control

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35
Q

What is a big challenge in clinical trials?

A

recruiting sufficient participants

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36
Q

What are the main reasons people choose to participate in clinical trials?

A
  • to advance medicine
  • to help improve the lives of others
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37
Q

What is patent protection?

A

No other companies can make or sell that drug for 20 years (protected from generic drugs)

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38
Q

What is a generic drug?

A

same chemical, off-patent

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39
Q

What are the major parts of an animal cell for drug discovery?

A

Nucleus: contains DNA
Plasma membrane (outside of cell): drugs act on many protein found here

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40
Q

What is a gene?

A

found in chromosomes, a recipe that codes for proteins (proteins is what we design drugs against)

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41
Q

What does the genetic code do?

A

converts RNA information to amino acid information

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42
Q

What are proteins?

A

huge chains of amino acids that are elaborately folded. Their shape is important for functions

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43
Q

Why is the shape of a protein important?

A

when drugs tackle proteins, the interaction between the drug and the protein shape is important

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44
Q

What is the central dogma?

A

the flow of information (DNA - RNA - Proteins)

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45
Q

What is cancer?

A

diseases of uncontrolled growth, including spreading into normal tissues to other parts of the body

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46
Q

What is metastatic cancer?

A

Cancer that spreads

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47
Q

How does cancer spread?

A

It starts as a tumor and breaks through the skin and detaches

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48
Q

How is cancer organized?

A

tissue/organ of origin
mechanism of growth dysregulation

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49
Q

What are the ways growth control is lost in cancer?

A
  1. growth machinery on overdrive and these proteins aren’t listening to the external growth signals (it’s just always on), it might have working stoping signals, but it can’t keep up with the pace of dysregulated growth
  2. growing machinery is healthy, but it lacks stopping machinery.
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50
Q

How do growth control mechanisms get dysregulated?

A

mutations in DNA or DNA damage, leading to dysfunctional proteins

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51
Q

What are the possible outcomes of DNA mutation?

A
  1. no effect
  2. base pair change causes a change in amino acid sequence (may or may not change protein structure and function)
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52
Q

What are some causes of genetic change?

A

Heredity (got from parents)
Viruses (some change DNA)
UV radiation ( energy can damage DNA)
Smoking (chemicals bind to DNA causing mutation)
Chemicals
Cells dividing

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53
Q

How does age relate to cancer?

A

as you get older, DNA mutation accumulates over time. Cancer increases with age

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54
Q

How do cancer cells compare to healthy cells?

A

They have different DNA

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55
Q

What is an inherited mutation?

A

mutation present in the egg or sperm cells that formed the child

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56
Q

What is an acquired mutation?

A

doesn’t come from a parent, acquired sometime later

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57
Q

What type of mutation mainly occurs in cancer?

A

acquired mutations

58
Q

Why do some cancer drugs stop working?

A

Cancer is constantly evolving new mutations (cancer of today isn’t the same as later)

59
Q

What are the most modern cancer drugs?

A

Targeted therapy

60
Q

What is targeted therapy?

A

aimed at proteins that are only in cancer cells, or that are far more abundant in cancer cells (have a more healthy profile)

61
Q

What is an example of a targeted cancer drug?

A

Herceptin

62
Q

What does Herceptin do?

A

HER2 receptors send signals to grow the tumor, Herceptin turns the protein off

63
Q

What do white blood cells in the immune system do?

A

attack viruses and bacteria

64
Q

How can the immune system attack cancer?

A

if it recognizes cancer cells foreign

65
Q

What is innate immunity?

A

Immunity you’re born with

66
Q

What is acquired immunity?

A

Immunity you develop when you are exposed to micro-organisms (vaccines)

67
Q

What are some specialized types of immune cells?

A

t cells and B cells

68
Q

What are T cells?

A

they bind to foreign peptides
some can destroy foreign entities (viruses)
They can kill cancer cells

69
Q

What are B cells?

A

can create antibodies, which bind to the surfaces of foreign cells
Can create antibodies against surface proteins on cancer cells

70
Q

How do cancer cells evade the immune system?

A
  1. The genetics of the cancer result in a cancer cell that can’t be recognized by the immune system
  2. cancer cells might have proteins on the surface that turn off the immune system
  3. the effect might be indirect - cancer cells change the healthy cells in the tissue around the tumor (alters how the immune system deals with the cancer cell)
71
Q

What is immunotherapy?

A

it helps our immune system to act better against cancer, by counteracting the way cancer cells avoid being destroyed

72
Q

What is an example of a genetic disease?

A

sickle cell disease

73
Q

What kind of protein structure is hemoglobin?

A

quaternary structure

74
Q

How does the structure of hemoglobin dictate its function?

A

the four subunits cooperate so that the complex can pick up more oxygen in the lungs and release it into the body

75
Q

What is hemoglobin made up of?

A

alpha globin and beta globin

76
Q

What is beta-thalassemia?

A

an inherited blood disorder in which the body doesn’t make as much beta-globin as it should

77
Q

How do sickle cells differ from healthy cells?

A

Sickle cells: altered beta-globin proteins, that stick together to form long fibers. The red blood cells carry oxygen but the fibers make them stiff and misshapen

Healthy: beta-globin combined with alpha-globin. Making red blood cells round and flexible

78
Q

How was sickle cell treated in first in human?

A

Deidra Williams - receives a hematopoietic stem cell transplant from a family member. They removed her own hematopoietic stem cells (chemo) and delivered a family member’s stem cells, allowing them to take over the production of red blood cells

79
Q

What causes sickle cell disease?

A

mutation in the HBB gene (hemoglobin subunit beta gene)

80
Q

What are some bad drug targets?

A
  • DNA and RNA (hard-to-find chemicals that will bind strongly)
  • Lipids (floppy molecules that can’t have interactions with small chemicals)
  • Carbohydrates (toxic)
81
Q

What are the main types of drug targets for current drugs?

A
  • Enzymes (protein machines that do work)
  • Receptors (proteins involved in communicating signal)
82
Q

What happens when a substrate binds to an enzyme?

A
  • 1 chemical (substrate) binds to the active site
  • enzyme changes shape when substrate binds
  • substrate is cut in half
  • 2 products leave the active site (chemical reaction)
83
Q

How can an enzyme undergo a reaction?

A

it is facilitated by amino acid residues of the enzyme

84
Q

What’s an example of binding a protein to a non-human protein?

A

COVID has an enzyme (protease) and the enzyme can be turned off with an inhibitor

85
Q

Where are receptors found?

A

on the surface or inside the cell

86
Q

What is a ligand?

A

it’s the chemical messenger that fits onto the receptor

87
Q

What are the different kinds of receptors?

A
  1. receptor inside the cell, drug needs to have chemical properties to enter the cell
  2. membrane-bound receptor, drug interacting (ligand on the outside), chemical conversion on the inside of cell
  3. membrane-bound receptor, drug interacting (ligand on the outside), receptor is bound to a protein on the inside, with an enzyme converting a substrate into 2 products
  4. Ion channel, membrane-bound receptor (like a tube), drug binds on the outside (changing the shape)
  5. membrane-bound receptor, drug interacting (ligand on the outside), once bound it might move downstream and interact with other receptors
88
Q

What is cell signaling?

A

a chain of molecular events producing communication

89
Q

What happens during cell signaling?

A
  1. Reception: ligase binds to the receptor
  2. Signal transduction: signal is being relayed (second messenger)
  3. Response: Second messengers relay their own cellular responses
90
Q

What is a drug-receptor interaction model?

A

Drug + receptor <—> drug-receptor complex (when the drug is bound to the receptor)

This leads to an effect

91
Q

What is pharmacodynamics?

A

branch of pharmacology concerned with the effects of drugs and the mechanism of their action

92
Q

How can a receptor have an altered physiological response?

A

if a drug binds to the receptor and it cannot interact with the ligase

93
Q

What is the premise of target identification?

A

a macromolecule (often protein) exists that is central to the disease mechanism

If we change the protein with a drug we will improve our health status

94
Q

What is genomics?

A

study of all the genes in the genome

95
Q

What is genetics?

A

the study of a single gene in isolation

96
Q

What types of field of study is target identification?

A

genomics

97
Q

How are drug targets identified?

A

with the use of genomes due to lower complexity

98
Q

What are genome-wide association studies?

A

Comparing the genomes of a group of individuals with a medical condition to a group of healthy individuals. Trying to determine the genes with higher frequency (identify SNPs) in people with disease than healthy

99
Q

What are cancer genome studies?

A

comparing the genome of cancers to the healthy material of healthy people. This reveals possible drug targets

100
Q

What is the challenge with cancer genome studies?

A

too many possible targets

101
Q

What are examples of model organisms?

A
  • budding yeast
    -zebra fish
    -roundworm
  • fruit flies
  • yeast
    -mice
  • nematode
102
Q

What are some advantages of using zebrafish?

A
  1. proteins similar to humans
  2. similar drug metabolism (getting rid of the drug)
  3. similar receptors in drugs
  4. similar physiology (heart rate)
103
Q

What is a single nucleotide polymorphism?

A

(SNPs) substitution of one base for another at a single site

sites of variation in our genomes

104
Q

What are some things SNP can account for?

A

differences in
- apperence
- risk for disease
response to drug

Many SNP have no effect

105
Q

What are some single-cell diseases?

A
  • cystic fibrosis
  • TaySaks
  • sickle cell anemia
106
Q

What are some multiple gene diseases?

A
  • diabetes
  • schizophrenia
  • autism
  • obesity
107
Q

How are SNPs associated with Type 2 diabetes?

A

SNPs have been associated with an increased risk of diabetes

108
Q

How does the personal genome project do?

A

sequence many people and correlate sequence to health status

109
Q

How is a target drug validated?

A

by validating in model organisms

110
Q

How do you measure where a drug is effective in a cell culture?

A

behaviors that can be measured in cells (cell growth, cell death)

111
Q

What are 2D cultures?

A

cells in a culture dish

112
Q

What are 3D organoids?

A
  • multiple cell types, cell-cell interactions
113
Q

What is a microphysiological system?

A
  • organoid technology
114
Q

What is translational medicine?

A

study of disease mechanisms and processes with a focus on application

115
Q

What are the 3 R’s principles in animal research?

A
  • replacement (can this be avoided)
  • reduction (fewer animals)
  • refinement (minimize pain)
116
Q

What are mouse models with human tumors called?

A

Xenograft mouse models

117
Q

What is the cause of DIPG?

A

(a genetic disease) the ACVR1 receptor has mutated in childhood brain cancer. The mutant has different signaling properties making it grow uncontrollably

118
Q

What is ALS?

A

progressive and fatal disease that causes motor neurons to break down

119
Q

What is an example of translational medicine?

A

Mice models getting an ACE2 receptor to mimic the binding receptor for COVID. Then vaccines were tested on the mice

120
Q

What is thalidomide?

A

1950s used for morning sickness
It causes birth defects in some children whose mothers took the drug during pregnancy

121
Q

When were changes in drug safety laws made?

A

1962 drug manufacturers required to prove scientifically that the drug was safe and effective

122
Q

What does the FDA require for drug safety testing?

A
  • test in two species (one rodent, non-rodent)
  • Longer exposure to the drug
  • Higher dosing
123
Q

How is toxicity tested?

A
  • death of cells in a dish
  • pathology (looking for shapes under a microscope)
  • Looking at blood/ urine samples
124
Q

What does Botox do?

A

Kills the nerves and muscle cells to communicate (making it so a person can’t show wrinkles)

125
Q

What is an in vivo test?

A

in a living organism

126
Q

What is an in vito test?

A

in glass (cell cultures)

127
Q

What causes toxicity?

A

may be caused by the drug or by one of its metabolites

128
Q

What is ames assay?

A

in vitro mutagenesis test
test for the carcinogenic potential of a compound (will it cause cancer)

129
Q

How would an ames assay be set up?

A
  • a genetically modified bacteria (grows in soup)
  • it will only grow if there is a specific enzyme (Histidine)
  • if the DNA mutates, then it can change to being able to grow in a histidine-free environment
130
Q

What would happen if an ames assay resulted in a culture dish full of bacteria?

A

stop testing that drug because it is causing DNA mutations

131
Q

What are the target organs for toxicity?

A

liver, kidney, heart, lung, brain, reproductive, skin

132
Q

How is testing done for reproductive toxicity?

A

sperm morphology and motility

133
Q

What’s the main reason for drug being pulled from the market?

A

cardiotoxicity

134
Q

What is a biomarker?

A

something that can be objectively measured (someone’s temperature, blood pressure)
assigning a number

135
Q

What are indirect biomarkers?

A

Something measured outside the body to figure out the internal state

136
Q

How can liver toxicity be measured?

A

A healthy liver has liver-specific enzymes that are contained in the liver. Liver toxicity causes liver cells to burst and release these enzymes, which are taken up in blood and can be detected in a blood test

137
Q

What are some important ethical values?

A
  • justice (fairness and equality)
  • nonmaleficence (do no harm)
  • Beneficence (done to benefit others)
  • nonexploitation (don’t take advantage)
  • respect
  • autonomy (respect a patient’s right to make decisions regarding his medical care)
138
Q

What are the goals of clinical research?

A
  • develop knowledge to improve health
  • develop knowledge to increase understanding of human bio
139
Q

What were some documents for ethics based on?

A

Nuremberg code (WW2)
Declaration of Helsinki (fills in Nuremberg gaps)

140
Q

What are the guidelines for ethical research?

A
  • value (be meaningful)
  • scientific validity (design of experiment)
  • fair subject selection
  • favorable risk-benefit ratio
  • independent review
  • informed consent
  • respect
141
Q

Why should value be an ethical requirement?

A
  • responsible use of finite resources
  • avoidance of exploitation
142
Q

What are some key components to a research ethics board (REB) application?

A
  • Recruitment
  • consent
  • balancing risk vs benefit in research
  • confidentiality
  • conflicts of interest
143
Q

What is on a consent form?

A
  • contact info
  • clearly defined purpose
  • risks and benefits
  • statement of voluntary consent
  • summary and signatures
  • updated version
144
Q

What was the case study about ethical research?

A

1990 anti-emetic (anti-vomiting for chemo patients) was developed. It was a placebo-controlled trial.

145
Q

How were the ethics in the ethical research case study evaluated?

A
  • value (placebos were not the standard of care at the time)
  • scientific validity (more valid would be new drug + chemo and old drug + chemo, since they were improving an already existing drug)
  • risk-benefit ratio (they anticipated the vomiting was going to be so sever they needed a rescue drug)

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