A Transgenic Growth Factor for Therapeutic Use Secreted from a Human Embryonic Stem Cell Line

In this study, cell therapy is not the final goal. Rather, the therapeutic product will be a transgenically-expressed Growth Factor secreted by human embryonic stem cells into the culture medium and subsequently purified before it is injected into patients.

Because the final material is intended for human application, the research establishment requires a licence from the Human Tissue Authority (HTA). As the stem cells are initially isolated from human blastocysts, the researchers require a licence form the Human Fertilisation and Embryology Authority (HFEA), a Favourable Opinion from a Research Ethics Committee (REC) and approval for the local National Health Service Research and Development (NHS R&D) office.

Once a cell line has been established from the isolated embryonic stem cells and as part of the HFEA conditions of its licence, the investigators should seek approval from the Steering Committee of the UK Stem Cell Bank and deposit a sample of their line at the bank.

The stem cells are subsequently to be modified with an adenoviral vector with a transgene encoding a human Growth Factor. As the cells are to be genetically modified, a notification to the Health and Safety Executive (HSE) is required.

The genetically modified stem cells themselves do not form any part of the final study product. Instead, the transgenic Growth Factor will be secreted by the cells and subsequently purified from the cell culture medium. Although this Growth Factor product itself will not be licenced, its manufacture and processing via the stem cell line still requires a Good Manufacturing Practice (GMP; See Glossary) licence from the Medicines and Healthcare products Regulatory Agency (MHRA).

Any clinical testing of the Growth Factor product requires approval from the MHRA, the local National Health Service Research and Development (NHS R&D) Office and a Favourable Opinion from a REC, in the standard way, as with any other type of clinical trial. (See the 'Clinical Trials Toolkit')

To see the Regulatory Route Map for this project, click on the route code M-98318. Alternatively, to see a Project View of the Regulatory Route, click on P-98318.