Pharmsci 101 Exam 1 with Complete
Solutions
What is a drug? - ANS-- A chemical introduced to the body to exert an effect
- Pharmaceutical science POV: drugs are used to combat disease (hypertension,
diabetes, cancer, etc.) or improve an individual's wellbeing (nutrients).
How do drugs act? - ANS-- Mostly on human targets: receptors, enzymes, hormones
and factors, ion channels, nuclear receptors, and nuclear receptors
- Majority of drugs (3/4ths) act on receptors and enzymes
What is a receptor? - ANS-- A macromolecule where a ligand binds causing a
physiological effect.
- Transfers a chemical signal from outside the cell to inside the cell --> cascade of
events --> augmented cell signaling
Example of a receptor - ANS-The ligand epinephrine binds to the adrenergic receptor,
causing an increase in blood pressure or bronchodilation (opening of airways)
Agonists - ANS-Mimic shape of ligand/neurotransmitter, causing an increase in drug
action/effect
Example of agonists - ANS-Betamethasone: glucocorticoid agonist - anti-inflammatory
Morphine: opioid agonist - relieves pain
Ventolin: adrenergic agonist - for asthma
Antagonists - ANS-Bind to receptors but don't activate them, which blocks the function
of a ligand/neurotransmitter
Example of an antagonist - ANS-Propanolol: adrenergic antagonist - antihypertensive
What is an enzyme? - ANS-- Biological catalysts that act on a substrate inside the body
- May break down an important substance or produce a chemical that causes a
physiological effect
How does a drug act at an enzyme? - ANS-Most drugs that act on an enzyme will inhibit
it
Examples of drugs that act on an enzyme - ANS-Aspirin: COX inhibitor - analgesic
Captopril: ACE inhibitor - antihypertensive
Viagra: PDE5 inhibitor - vasodilation
,Other targets - ANS-- Drugs that affect other modes of communication, such as ion
channels (which allow ions in and out)
- Drugs that act like hormones or affect their concentration
- Drugs that act at the DNA/RNA level
- Drugs that affect transporters, which allow chemicals back into neurons
Drugs that act at a transporter or channels - ANS-- Prozac: blocks serotonin uptake -
antidepressant
- Rogaine: opens potassium channels - antihypertensive and hair growth
Do all drugs act on a human target? - ANS-No, antibiotics and antacids are examples of
drugs that don't.
Druggability of targets - ANS-A druggable target is one where a drug can reach, bind
and produce an effect.
How do we know if a target is druggable? - ANS-If it's similar to one known to be
druggable, or through techniques such as X-ray crystallography.
What is a good drug? - ANS-Must be efficacious, potent, and safe.
Therapeutic index - ANS-The ratio between the toxic and therapeutic concentrations of
a drug
TI = TD50/ED50
Drugs with low therapeutic index - ANS-- Must be careful because as much as double
the dose could be very toxic
- Examples: Digoxin, Theophylline
Drugs with higher therapeutic index - ANS-Generally safer
Magic bullet - ANS-a term coined by Paul Ehrlich to describe the ideal function of a drug
compound: to kill the pathogen but leave the host unharmed
Magic gun - ANS-Delivery system must be optimal and only deliver drug to one specific
target
Active Pharmaceutical Ingredient (API) - ANS-The biologically active component in a
drug.
Pharmacology - ANS-Branch of medicine and biology concerned with the study of drug
action. Deals with the uses and especially the effects of drugs.
1906 Pure Food and Drug Act (Wiley Act) - ANS-prohibited interstate commerce of
adulterated and misbranded food and drugs.
, Medicinal chemistry - ANS-Design and development of pharmaceutical compounds.
Pharmaceutical sciences - ANS-- group of interdisciplinary areas of study involved with
the design, action, delivery, disposition and use of drugs
- Turning drugs into drug products
Clinical pharmacy and translational sciences - ANS-- the area of pharmacy concerned
with the science and practice of rational medication use
- Drug product approval --> clinical use of drug product (drug administration)
Pharmacology process - ANS-Molecules --> drugs --> drug products --> drug product
approval --> clinical use of drug product (drug administration) --> drug molecules
delivered to site of action --> pharmacological activity
Kefauver-Harris Amendment of 1962 - ANS-• Also known as the "Drug Efficacy
Amendment".
• required NDAs to contain evidence that a new drug was effective as well as safe, and
that benefits of the drug outweighed its known risks.
Drug development process - ANS-1) Identify and validate the biological mechanism
behind a disease
2) Identify molecules that are active on the target protein
3) Identify classes of molecules that have the potential to become pharmaceuticals
4) Optimize the molecules' properties so that they're safe and effective
5) Candidate drugs: selection of substances with optimized properties for further
development
6) Preclinical trials in order to document that the compound is safe for entry into human
trials (in vitro and in vivo)
7) Investigational New Drug Application (IND) is the green light to explore drug in
humans
8) Phase 1: checking for safety, 20-100 volunteers
9) Phase 2: checking for efficacy, 100-500 patients - how well does the drug work?
10) Phase 3: confirm results, 1000-5000 patients - drug must be safe, gets compared
with the current "gold standard" treatment. This is the most expensive stage
11) New Drug Application (NDA) submitted (or Biologics License Application)
12) FDA review/Phase 4 trials: safety surveillance in "real life" patients
13) After getting the drug product package insert, need to set up spontaneous reporting
system (pharmacovigilance), reporting all serious un-labeled events. Annual reports are
made of all adverse events (adverse drug reactions, drug-drug interactions)
Radiation therapy - ANS-• Effective non-surgical treatment
• High doses of radiation directed at tumor
• Confined to area of tumor
• Palliation: shrinking of inoperable tumors for comfort
Solutions
What is a drug? - ANS-- A chemical introduced to the body to exert an effect
- Pharmaceutical science POV: drugs are used to combat disease (hypertension,
diabetes, cancer, etc.) or improve an individual's wellbeing (nutrients).
How do drugs act? - ANS-- Mostly on human targets: receptors, enzymes, hormones
and factors, ion channels, nuclear receptors, and nuclear receptors
- Majority of drugs (3/4ths) act on receptors and enzymes
What is a receptor? - ANS-- A macromolecule where a ligand binds causing a
physiological effect.
- Transfers a chemical signal from outside the cell to inside the cell --> cascade of
events --> augmented cell signaling
Example of a receptor - ANS-The ligand epinephrine binds to the adrenergic receptor,
causing an increase in blood pressure or bronchodilation (opening of airways)
Agonists - ANS-Mimic shape of ligand/neurotransmitter, causing an increase in drug
action/effect
Example of agonists - ANS-Betamethasone: glucocorticoid agonist - anti-inflammatory
Morphine: opioid agonist - relieves pain
Ventolin: adrenergic agonist - for asthma
Antagonists - ANS-Bind to receptors but don't activate them, which blocks the function
of a ligand/neurotransmitter
Example of an antagonist - ANS-Propanolol: adrenergic antagonist - antihypertensive
What is an enzyme? - ANS-- Biological catalysts that act on a substrate inside the body
- May break down an important substance or produce a chemical that causes a
physiological effect
How does a drug act at an enzyme? - ANS-Most drugs that act on an enzyme will inhibit
it
Examples of drugs that act on an enzyme - ANS-Aspirin: COX inhibitor - analgesic
Captopril: ACE inhibitor - antihypertensive
Viagra: PDE5 inhibitor - vasodilation
,Other targets - ANS-- Drugs that affect other modes of communication, such as ion
channels (which allow ions in and out)
- Drugs that act like hormones or affect their concentration
- Drugs that act at the DNA/RNA level
- Drugs that affect transporters, which allow chemicals back into neurons
Drugs that act at a transporter or channels - ANS-- Prozac: blocks serotonin uptake -
antidepressant
- Rogaine: opens potassium channels - antihypertensive and hair growth
Do all drugs act on a human target? - ANS-No, antibiotics and antacids are examples of
drugs that don't.
Druggability of targets - ANS-A druggable target is one where a drug can reach, bind
and produce an effect.
How do we know if a target is druggable? - ANS-If it's similar to one known to be
druggable, or through techniques such as X-ray crystallography.
What is a good drug? - ANS-Must be efficacious, potent, and safe.
Therapeutic index - ANS-The ratio between the toxic and therapeutic concentrations of
a drug
TI = TD50/ED50
Drugs with low therapeutic index - ANS-- Must be careful because as much as double
the dose could be very toxic
- Examples: Digoxin, Theophylline
Drugs with higher therapeutic index - ANS-Generally safer
Magic bullet - ANS-a term coined by Paul Ehrlich to describe the ideal function of a drug
compound: to kill the pathogen but leave the host unharmed
Magic gun - ANS-Delivery system must be optimal and only deliver drug to one specific
target
Active Pharmaceutical Ingredient (API) - ANS-The biologically active component in a
drug.
Pharmacology - ANS-Branch of medicine and biology concerned with the study of drug
action. Deals with the uses and especially the effects of drugs.
1906 Pure Food and Drug Act (Wiley Act) - ANS-prohibited interstate commerce of
adulterated and misbranded food and drugs.
, Medicinal chemistry - ANS-Design and development of pharmaceutical compounds.
Pharmaceutical sciences - ANS-- group of interdisciplinary areas of study involved with
the design, action, delivery, disposition and use of drugs
- Turning drugs into drug products
Clinical pharmacy and translational sciences - ANS-- the area of pharmacy concerned
with the science and practice of rational medication use
- Drug product approval --> clinical use of drug product (drug administration)
Pharmacology process - ANS-Molecules --> drugs --> drug products --> drug product
approval --> clinical use of drug product (drug administration) --> drug molecules
delivered to site of action --> pharmacological activity
Kefauver-Harris Amendment of 1962 - ANS-• Also known as the "Drug Efficacy
Amendment".
• required NDAs to contain evidence that a new drug was effective as well as safe, and
that benefits of the drug outweighed its known risks.
Drug development process - ANS-1) Identify and validate the biological mechanism
behind a disease
2) Identify molecules that are active on the target protein
3) Identify classes of molecules that have the potential to become pharmaceuticals
4) Optimize the molecules' properties so that they're safe and effective
5) Candidate drugs: selection of substances with optimized properties for further
development
6) Preclinical trials in order to document that the compound is safe for entry into human
trials (in vitro and in vivo)
7) Investigational New Drug Application (IND) is the green light to explore drug in
humans
8) Phase 1: checking for safety, 20-100 volunteers
9) Phase 2: checking for efficacy, 100-500 patients - how well does the drug work?
10) Phase 3: confirm results, 1000-5000 patients - drug must be safe, gets compared
with the current "gold standard" treatment. This is the most expensive stage
11) New Drug Application (NDA) submitted (or Biologics License Application)
12) FDA review/Phase 4 trials: safety surveillance in "real life" patients
13) After getting the drug product package insert, need to set up spontaneous reporting
system (pharmacovigilance), reporting all serious un-labeled events. Annual reports are
made of all adverse events (adverse drug reactions, drug-drug interactions)
Radiation therapy - ANS-• Effective non-surgical treatment
• High doses of radiation directed at tumor
• Confined to area of tumor
• Palliation: shrinking of inoperable tumors for comfort
