New Drug Technologies

Question 1

What are Mabs?

Answer 1

monoclonal antibodies

Question 2

What are the three types of Mabs?

Answer 2

chimeric-murine/human
humanized-95% human
fully human

Question 3

What do Mabs bind to?

Answer 3

bind to receptor or ligand

Question 4

What is the nomenclature for Mabs?

Answer 4

suffix: mab
Middle: two letters indicating source of antibodies

Question 5

How do you know if a Mab is sourced from humans?

Answer 5

has “mu”

Question 6

What is an example of a Mab and what does it do?

Answer 6

Bevacizumab-directed at VEGF and cuts off blood supply to tumors

Question 7

What are antibody drug conjugates?

Answer 7

Mab joined to another drug to better target the cell

Question 8

What is an example of an antibody drug conjugate and what does it do?

Answer 8

Trastuzmab entansine-binds HEP-2 which causes growth and with antibody the drug can actually enter the cell

Question 9

What are the limitations of Mabs?

Answer 9

injection only, possibility for immunological reactions and very very expensive

Question 10

What are fusion proteins?

Answer 10

fusing receptor to parts of an antibody

Question 11

What is an example of a fusion protein and what does it treat?

Answer 11

etanercept-binds TNF and helps rheumatoid arthritis?

Question 12

What are the two types of gene therapies talked about in class?

Answer 12

CAR-T and antisense oligonucletodies

Question 13

How does CAR-T gene therapy work and what is its main target?

Answer 13

Remove T-Cells from patient and get them to express the CD19 receptor, clone them and reinject it, targets tumors

Question 14

What is one of the potential issues with CAR-T therapy and how is it fixed?

Answer 14

increased cytokine release and fixed by inhibiting IL-6

Question 15

What is an example of a CAR-T drug and what kind of tumor does it target?

Answer 15

tisagenleucel-targets blood borne tumors

Question 16

How do first gen and second gen antisense olgionucelotides work?

Answer 16

first gen: bind to sense strand and prevents translation
second: binds to mRNA which then activates the ribonucleases that degrade mRNA, and then can bind other mRNAs

Question 17

What are two examples of second gen ASOs and what do they treat?

Answer 17

mipomensin-lowers Apolipoprotein B and thus lowers cholesterol
nusinersen: spinal muscular atrophy

Question 18

How do siRNAs work?

Answer 18

dsRNA is cleaved by DICER and then separated by RISC/AGO2 and then prevents binding like other ASOs

Question 19

What is an e xample of an siRNA drug and what does it treat?

Answer 19

Patisiran-treats hATTR which is mutation in the TTR gene where it deposits amyloid into tissues

Question 20

What is an aptamer?

Answer 20

string of nucleotides that bind proteins-first discovered in viruses

Question 21

What is an example of a aptamer and what does it treat?

Answer 21

Pegaptinib-binds VEGF and used for age related macular degeneration that causes leaky blood vessels in the eyes leading to blindness

Question 22

What is a potential use for CRISPR-Cas9 in drugs?

Answer 22

slice out PCKS9 to cure high cholesterol

Question 23

What is the purpose of having biosimilar Mabs?

Answer 23

come at a much cheaper price, but not exactly identical

Question 24

What are the four types of regenerative medicines talked about in this class?

Answer 24

growth factor receptors, stem cells, nanotechnology and cell therapy

Question 25

Describe the premise of growth receptor therapy

Answer 25

using the receptors in the body to grow new tissues

Question 26

Where are two areas in the body where we have seen 'success' with growth receptor therapy?

Answer 26

angiogenic growth factor-new coronary vessels in periphery wound healing-diabetic foot ulcers treating with platelet derived growth factors which stimulates smooth muscle, ECM, collagen, fibroblasts, neutrophils and monocytes

Question 27

What drug helped with the foot ulcers?

Answer 27

becaplemin

Question 28

How as of right now is growth receptor stimulation being administered and why?

Answer 28

directly into tissue, but mixed results, IV risk of cancer and metabolism

Question 29

What is the main concern with growth receptor stimulation?

Answer 29

tumor formation and existing tumor growth

Question 30

What is the premise behind cell therapy?

Answer 30

healthy cells are harvested, cloned and reinjected

Question 31

What is the name of the drug that helps with cartilage reformation and describe the process.

Answer 31

carticel-autologous chondrocytes are harvested and grown to replenish cartilage

Question 32

What do azficel and apligraf do?

Answer 32

azficel-kertainocytes for wrinkles apligraf- fibroblasts for skin substitutes

Question 33

What are the three forms of stem cells?

Answer 33

fetal embryonic mesenchymal

Question 34

Where are mesenchymal stem cells sourced from?

Answer 34

fat, bone marrow, skin, periodontal

Question 35

What are special about MSCs and where has some success been shown?

Answer 35

immune modulating effects-darvastrocel has been used for decreasing inflammation and bowel repair in peoples with Crohns

Question 36

Where has there been success with stem cell therapy?

Answer 36

wound healing and visual impairments, varied success in cardiovascular and parkinson's

Question 37

What are the main issues with stem cell treatment?

Answer 37

ethics and delivery

Question 38

What is the premise behind nanotechnology?

Answer 38

using technology for better drug delivery

Question 39

Where are two applications where nanotechnology is already seen?

Answer 39

dexcoms pills with cameras to scope out GI tract deoxorubicin- uptake where cancer cells only recieved drug

Question 40

What is one form of nanotechnology and what is its downfall?

Answer 40

micelle like pill so it can be absorbed-however the polyethylene groups are not always stable

Question 41

What was an example of nanotechnology where they were able to cross the BBB?

Answer 41

used neutrophils