Therapeutic use of a Foetal Stem Cell Line Expandable in Culture via a Doxycycline-Responsive RAS

In this clinical study, stem cells are initially isolated from the central nervous system of a terminated foetus, then subsequently genetically modified using an integrating retrotransposon encoding the activated Ras oncogene under the control of a doxycycline-responsive promoter. These cells can thus be expanded in laboratory culture medium containing doxycycline and subsequently purified, ahead of introduction into patients.

As the stem cells are intended for human application, the research establishment requires a licence from the Human Tissue Authority (HTA). Genetic modification of the cells in the laboratory requires notification to the Health and Safety Executive (HSE). Following dialogue with the Medicines and Healthcare products Regulatory Agency  (MHRA), the cells are tested under Good Laboratory Practice (GLP; See Glossary) in order to assess their safety and efficacy in animal hosts. This animal research also requires the approval of the Home Office Animal Licensing Inspectorate.

Before the clinical trial begins and in order to obtain sufficient quantities of cells, the line is expanded in culture under Good Manufacturing Practice (GMP; See Glossary) conditions. Such facilities must be approved by the Medicines and Healthcare products Regulatory Agency GMP inspectorate. Because the clinical trial involves the use of a somatic cell therapy medicinal product, approval by the MHRA Clinical Trials Unit is required. Favourable Opinion by the Gene Therapy Advisory Committee (GTAC) is also needed because the cell therapy has been derived from a Stem Cell Line (See Glossary). In addition, notification to the HSE of the clinical trial involving such a genetically-modified product is required. Approval of the local National Health Service Research and Development (NHS R&D) office is also necessary.

After sufficient quality, safety and efficacy data have been obtained in clinical trials, an application can be made to the European Medicines Agency (EMEA) to obtain Marketing Authorisation to use the therapy as a standard treatment.

To see the Regulatory Route Map for this project, click on the route code M-90590. Alternatively, to see a Project View of the Regulatory Route Map for this project, click on P-90590.