L4- Regulatory Perspective

Question 1

What are examples of advanced therapies?

Answer 1

♦ Gene therapy
♦ Somatic cell therapy
♦ Tissue engineering

Question 2

Why is a regulatory framework needed for advanced therapies?

Answer 2

They can cause detrimental effects and kill people instead of curing or treating them
♦ Vision loss occurred in patients who were injected with autologous adipose tissue derived ‘stem cells’ to treat AMD. (2017)
♦ Lesion occurred in spinal cord as a consequence of ‘stem cell tourism’.

Question 3

What are the questions that need to be considered when working on advanced therapies?

Answer 3

1. Contents of the tube
2. Treatment mode of action
3. Whether the treatment is a therapy or procedure
4. The Equipose Problem
5. Conflict of interest and informed consent

Question 4

How are the contents of the therapy monitored?

Answer 4

♦ Cell banking and manufacture
♦ Testing in preclinical models
♦ Formulated drug product to patient

Question 5

How is the mechanism of the treatment investigated?

Answer 5

♦ Working out the mode of action
♦ Testing with a potency assay
♦ Testing with a release assay

Question 6

What are the possible modes of action?

Answer 6

♦ Immunomodulation
♦ Modulation of endogenous stem cells
♦ Neurotrophic mechanisms

Question 7

How is the science gap addressed?

Answer 7

Where good functional assays are not available we can at least be sure that the cell preparations are clean and pure and consistent.

Question 8

What is the equipoise problem?

Answer 8

Genuine uncertainty in the expert medical community about whether a treatment will be beneficial or not