Therapeutic Use of Dopaminergic Neurons Purified from Chemically-Differentiated Human Embryonic Stem Cells

In this clinical study, stem cells are initially isolated from human blastocysts. Once a human embryonic stem cell line is established, the investigators will define culture media which promote the differentiation of stem cells into dopaminergic neurons. The efficacy and safety of these purified neurons will be assessed in animal hosts. Subsequently, the stem cell-derived neurons will be tested in patients suffering from Parkinson's Disease.

Because the stem cells to be used in this study are intended for human application, the research establishment requires a licence from the Human Tissue Authority (HTA). Derivation of the stem cell line requires a licence form the Human Fertilisation and Embryology Authority (HFEA), a Favourable Opinion from a Research Ethics Committee (REC) and the approval of the local National Health Service Research and Development (NHS R&D) office.

Once a cell line is established from the isolated embryonic stem cells and as part of the HFEA conditions of its licence, the investigators should subsequently seek approval from the Steering Committee of the UK Stem Cell Bank to deposit the line there.

Following dialogue with the Medicines and Healthcare products Regulatory Agency (MHRA), the cells are tested under Good Laboratory Practice (GLP; See Glossary) conditions to assess their safety and efficacy in animal hosts. This animal research requires the approval of the Home Office Animal Licensing Inspectorate.

Before a clinical trial can begin, the stem cells are expanded in culture under Good Manufacturing Practice (GMP; See Glossary) conditions. Facilities are approved by MHRA GMP inspectors. Because the clinical trial involves a somatic cell therapy medicinal product, approval by the MHRA Clinical Trials Unit is required. Because the trial involves a cell therapy derived from a Stem Cell Line (see Glossary), a Favourable Opinion by the Gene Therapy Advisory Committee (GTAC) is required. Approval of the local National Health Service Research and Development (NHS R&D) office is also needed.

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-89595. Alternatively, to see the Project View of this Regulatory Route, click on P-89595.