week 5 part 2

Question 1

Where is technology going from?

Answer 1

1. Rodent
2. Chimeric
3. Humised
4. Human

more human like and less immunogenic

Question 2

What is haemophilia caused . by?

Answer 2

Failure to produce certain proteins required for blood clotting

1. Factor VIII (haemophila A)
2. Factor IX (haemophila B)

Question 3

How many Haemophila A be restored?

Answer 3

Infusion of recombinant FVIII

Question 4

How does the body make inhibitory antibodies?

Answer 4

If you are haemophila A type and you’ve never made factor 8

You’ve been given recombinant protein

Question 5

Why do we change treatment?

Answer 5

To give pre-formed complexes to help the cascade

Question 6

What are Lysosomal storage disorder?

Answer 6

1. Inherited disorder resulting from a lack of specific enzymes that break down certain lipids (fats) or carbohydrates (sugars) in body cells

Question 7

What disrupts normal function and cause lysosomal storage disorder?

Answer 7

If a person does not have enough of one of these enzymes, the body cannot break down the fat or carbohydrate targeted by enzymes for recycling. These fats or sugars accumulate in cell lysosomes where enzymes are active

Question 8

What does Enzyme replacement therapy involve?

Answer 8

Intravenous infusion to correct the underlying enzyme deficiency that causes symptoms of Gaucher disease

Question 9

What is the enzyme replacement therapy drug for Gaucher’s?

Answer 9

Cerezyme

Question 10

What is the enzyme replacement therapy drug for Fabry’s disease?

Answer 10

Fabrazyme

Question 11

What is Myozyme used to treat?

Answer 11

Patient who have Pompe disease

A rare inherited disorder

Question 12

What do patients with pome disease do not have enough of?

Answer 12

Enzyme - alpha glucosidase

Breaks down sugar stored as glycogen into glucose that can be used for energy by the body’s cells

Question 13

What is the first enzyme replacement therapy to specifically treat the underlying cause of MPS1?

Answer 13

Aldurazyme (Iaronidase)

Question 14

What is Infliximab?

Answer 14

The active ingredient in REMICADE

1. A chiemric IgG1K monoclondal antibody specific for human tumour necrosis factor alpha (TNFalpha)

Question 15

What is the molecular weight of Infliximab?

Answer 15

149.1 kilodaltons

Question 16

What is Infliximab produced by?

Answer 16

Recombinant cell line cultured by continuous perfusion and is purified by a series of steps that includes measures to inactivate and remove viruses

Question 17

What did patients with tuberculosis frequently presented with?

Answer 17

Disseminated or extrapulmonary disease

Question 18

What should patients be tested for before Remicade use and during therapy?

Answer 18

Latent tuberculosis

Question 19

When should treatment for latent infection be initiated?

Answer 19

prior to REMICADE use

Question 20

What is Alemtuzumab used for?

Answer 20

MS

Drug is given 5 days in a row for the first ear

Then exactly a year later - 3 days in a row

In subsequent years -3 more days

Question 21

What are the cost for Alemtuzumab?

Answer 21

First year - around 35,000

Second year - 21,000

Question 22

What is Lemtrada?

Answer 22

CD52-directed cytolytic monoclonal antibody indicated for the treatment of patients with relapsing forms of multiple sclerosis (MS)

Question 23

What does Alemtuzumab bind to?

Answer 23

Cb52 which is on B cells or NK cells

Depletes your lymphocytes

come of treatment: secondary autoimmune conditions

Deplete CD4 regulatory cells

Question 24

Alemtuzumab

Answer 24

a monoclonal antibody that targets an antigen known as CD52, a common antigen found on B and T cells (part of the body’s immune system). When the alemtuzumab antibody attaches to the CD52 antigen, the body’s immune system is activated to destroy these targeted cells in the blood and bone marrow. Since the CD52 antigen is also present on healthy B and T cells, however, treatment will temporarily weaken your immune system and care must be taken to protect you from infection during treatment

Question 25

What is Adalimumab (Humira)?

Answer 25

is an anti-TNF drug. In rheumatoid arthritis and some other conditions, too much of a protein called TNF (tumour necrosis factor) is produced in the body, causing inflammation, pain and damage to the bones and joints. Anti-TNF drugs such as adalimumab block the action of TNF and so reduce this inflammation

Question 26

Where was anti-adalimumab detected?

Answer 26

76 patients (28%)

♣ Shows that 51 of 76 patients (67% of antiadalimumab antibody−positive patients) developed antiadalimumab antibodies during the first 28 weeks of treatment

Question 27

What is Natalizumab?

Answer 27

A humanized monoclonal antibody against the cell adhesion molecule alpha-4 integrin

Question 28

What is Natalizumab used in?

Answer 28

Treatment of multiple sclerosis and Chrohn’s disease

Question 29

How is Natalizumab administered?

Answer 29

Intravenous infusion every 28 days

Works by reducing the ability of inflammatory immune cells to attach to and pass through the cell layers lining the intestine and BBB

Question 30

Why was Natalizumab withdrawn from the market?

Answer 30

linked with three cases of the rare neurological condition progressive multifocal leukoencephalopathy (PML)

Question 31

Why didnt the FDA not withdraw Natalizumab from market?

Answer 31

Clinical benefit outweighs the risk involved

Question 32

What is Rituximab?

Answer 32

Targets the CD20 antigen on normal and malignant B cells

The body’s natural immune defences are recruited to attack and kill the marked B cells

Stem cells do not have the CD20 antigen

This allows healthy B-cells to regenerate after treatment

Question 33

How does Ocrelizumab work by?

Answer 33

Targeting a type of immune cell

This helps to reduce the immune response by stopping these cells from attacking and damaging myelin