GCD 2171 - Midterm 2

which animals make up 95% of research animals in biomed?

rats and mice

list 4 reasons why animals are used in biomed research

• humans and animals share many biological and physiological characteristics (e.g. immune systems).  

• they have a shorter life cycle than humans → animals can be studied throughout their life span and for generations

• scientists can easily control the environment around animals (diet, temperature, lighting)

• it is expected that medical treatments (e.g., stem cell treatments) first demonstrate safety and efficacy in animal models before they can be introduced in humans

what are alternative methods to animal use?

computer models, tissue/cell cultures, organs on chips

what does USDA stand for?

U.S. Department of Agriculture

true or false + correct the statement: the U.S. Food and Drug Administration (FDA) has set federal regulations for the care and use of animals in biomedical research

false; the USDA not FDA

what does the Animal Welfare Act do?

sets standards of care for research animals

who sets federal regulations for the care and use of animals in biomedical research

USDA

what does IACUC stand for?

Institutional Animal Care and Use Committee

what does IACUC do?

oversee research with animals + require researchers to justify their need for animals and define study design and procedures

true or false + correct the statement: once you file an animal protocol, you can start your animal experiments before an approval by IACUC

false; IACUC approval is required beforehand

list two key reasons for high quality of animal care and treatments

• the use of animals in research is a privilege

• a well-treated animal will provide reliable scientific results

true or false + correct the statement: drugs need not be tested in animals to receive FDA approval

true

what does the FDA stand for?

U.S. Food and Drug Administration

list four major sources of funding for biomed research

• government grants

• private foundation money

• private donation

• corporate funding

which type of funding is most important for biomed research?

government grants

true or false + correct the statement: the government funds research after it reaches pre-clinical development

false; before not after

what does NIH stand for?

National Institutes of Health

what does the NIH do?

provides the biggest financial support to biomedical research in the US

list the government’s 4 main roles/duties in supporting research and development

• provide funding

• encourage basic research and innovation

• regulate for safety

• restrict questionable practices

true or false + correct the statement: the use of non-human primates in research is strictly prohibited due to ethical reasons

false; not prohibited but regulated

what does NSF stand for?

National Science Foundation

what does the NSF do?

supports research and education in fundamental science and engineering, except for medical sciences

list 3 characteristics of private foundations

• disease-specific

• non-profit

• focus on research and patient care

what was the CIRM?

california-specifc institute made to support and advance stem cell research, but poor funding management and conflict of interests caused its dissolvation (however, voting in 2020 allowed it to get more funding → still active today)

true or false + correct the statement: some disease-specific private organizations focus on patient support and raising public awareness rather than financially supporting research on the disease they are interested in

true

what did the Leahy-Smith America Invents Act (AIA) do?

clarified that stem cells are patent eligible, but patent claims directed to or encompassing a human organism, including human embryos are prohibited

true or false + correct the statement: the AIA does not define the term “human organism”

true

true or false + correct the statement: stem cells are not patent eligible

false; they are eligible under the AIA (but hESCs may not be depending on the SCJ)

what did the Myriad Decision do?

ruled that cDNAs are patent eligible, but isolated DNAs are not

true or false + correct the statement: in what is called the Myriad decision, the Supreme Court ruled that isolated DNAs are not patent eligible

true

what does MTA stand for?

Material Transfer Agreement

what is an MTA?

a contract that governs the transfer of research materials between two organizations + defines the rights of the provider and the recipient with respect to the materials and any derivatives

true or false + correct the statement: federal law claims that life begins at conception

false; there is no federal law on when life begins

true or false + correct the statement: Americans have become less supportive of embryonic stem cell research in the past 15 years

false; they have become more supportive

what is the general Catholic & Orthodox stance on hESC use?

against all use

what is the general Protestant stance on hESC use?

mixed

what is the general Jewish stance on hESC use?

generally supportive

when do most Jewish people view ensoulment to begin?

after 40 days

what is the general Muslim stance on hESC use?

mixed

when do most Muslim people view ensoulment to begin?

not until 120 days

when do most Catholic & Orthodox people view ensoulment to begin?

at fertilization

what is the general Hindu stance on hESC use?

mixed

what is the general Buddhist stance on hESC use?

mixed

when do most Hindu people view ensoulment to begin?

3-5 months

when do most Buddhist people view ensoulment to begin?

at fertilization

how did the NIH interpret the Dickey Amendment?

federal funding can be used to study, but not to derive, hESC lines

how did Bush impact stem cell research?

he restricted (not banned) the number of hESC lines that could be used in federally funded research

true or false + correct the statement: you cannot make ESCs solely for research in the US

false; you can just not with federal funding

what does IRB stand for?

Institutional Review Board

what does the IRB do?

reviews and monitors biomedical research involving human subjects

list 5 examples of research that is ineligible for government funding

• deriving new stem cells

• using human fetal tissues

• cloning

• creating human-animal chimeras

• breeding animals with human germ lines

how many phases are there in clinical trials?

4

what does phase i of clinical trials assess?

safety, dosage range, side effects

what does phase ii of clinical trials assess?

effectiveness, further evaluate safety

what does phase iii of clinical trials asssess?

confirm effectiveness, compare to existing treatments, monitor side effects, define strategy that allows safe use

what does phase iv of clinical trials assess?

follow-up studies to collect more information on risks, benefits, and optimal use

how many subjects in phase i of clinical trials?

20-80

how many subjects in phase ii of clinical trials?

100-300

how many subjects in phase iii of clinical trials?

1000-3000

why do phase i clinical trials assess safety rather than effectiveness?

even if a treatment works in animals, it does not mean it will work in humans

list 2 risks of transplanting cells that include an undifferentiated pluripotent stem cell

teratoma formation and tissue rejection

list 4 reasons that the eye is advantageous for clinical trials for stem cell-based replacement therapy

• established procedures to make RPE cells from ESCs

• the eye is an "immune-privileged" location; tissue rejection is less likely

• the eye is a "closed" unit; even if tumors develop, tumor cells are less likely to escape

• undesired tumorigenesis can be easily monitored by eye exams

true or false + correct the statement: there are clinical trials that use cells derived from pluripotent human ESCs or iPSCs

true

what does the FDA do?

protects and promotes public health through regulatory programs on food, drugs, etc.

true or false + correct the statement: the FDA regulates “biological products”, which include human tissue and cellular products used in transplantation

true

why are drugs so expensive?

cost lies in research and development

list 3 reasons why drugs are so expensive

• extremely tedious and time consuming (can take 12-15 years)

• can cost up to $1 billion from research to market

• patents only valid for 20 years, leaves very narrow window for companies to recoup investment

true or false + correct the statement: in clinical trials, the most costly part is phase i trials

false; phase iii is most costly

what does EUA stand for?

Emergency Use Authorization

what can the FDA do under the EUA?

allow the use of unapproved medical products, or unapproved uses of approved medical products in an emergency

what is “off-label” use?

using an FDA-approved drug for an unapproved use

what does it mean if a drug is unapproved for use?

the FDA has not determined that the drug is safe and effective