Introduction

Question 1

What happens when we redesign natural hormones to make them into anti-diuretic drugs (reduce urine volume)?

Answer 1

They get longer half-life and improved pharmacokinetics.
E.g.: Natural hormone Vasopressin redesigned into the drugs Desmoprissin and Glypressin.

Question 2

What are pharmaceutical drugs based on large biomolecules & cells?

Answer 2

• Antibodies
• Cytokines
• Soluble receptors
• Aptamers
• CAR T cells
• Activated cells
• Vaccines
• Others

Question 3

What are ATMPs (Advanced Therapy Medicinal Products)?

Answer 3

ATMPs are medicines for human use with an active therapeutic substance based on at least one of the following:
- Technology to modify patient genome
- Recombinant nucleic acids or genes
- Substantially manipulated cells
- Cells intended for a different essential function in the patient versus the donor
- Engineered tissue

Question 4

What are CAR T cells?

Answer 4

Genetically modified T cells typically derived from the patient him-/herself. They carry artificial receptors that target the T cell to the cancer.

Question 5

How does CAR T cell therapy work?

Answer 5

1) Remove blood from the patient to get T cells.
2) Make CAR T cells in the lab. This is done by inserting the gene for CAR (Chimeric Antigen Receptor) into the T cell.
3) Then grow millions of CAR T cells on agar plates.
4) Infuse the CAR T cells into the patient.
5) CAR T cells then bind to cancer cells (by the Chimeric Antigen Receptors) and kill them.

Question 6

What about checkpoint inhibitors?

Answer 6

• They have the ability to unblock the immune system’s power by circumventing tumor’s ability to turn it off.
• They are able to transform untreatable cancers into chronic diseases e.g. melanoma (most serious type of skin cancer).
• Side-effects are dose-limiting and problematic.

It can block a tumor’s ability to avoid immune destruction and thereby allow the immune system to control the tumor and its development.

Question 7

What is the main benefit with long half-life?

Answer 7

It limits the need of large quantities of expensive drugs.

Question 8

What can you tell about anti-inflammatory drugs e.g. anti-TNF?

Answer 8

• TNF (Tumor Necrosis Factor) is an important component of inflammation, which in turn is an important part of protection against infectious diseases and an important component for the development of autoimmune diseases.
• Inhibition of TNF blocks inflammation.
• Anti-inflammatory drugs has antibodies and natural TNF-receptors as active components.
• Drugs exploits (utnyttjar) the properties of Fc-FcRN interaction to extend the half-life of soluble receptors.

Question 9

What causes autoimmune diseases and how to prevent it?

Answer 9

Over-reactive immunity may cause autoimmune disease, and soluble CTLA4 may down-regulate the power of immunity and resolve such disease.