Clinical trials of cell therapies
Cell therapy is therapy in which cells are administered to a patient. The cells may be given by iv
infusion, local injection or by surgery e.g. using cells immobilised on a scaffold. In autologous cell
therapy the patient receives back their own manipulated or processed cells which have been
previously collected from them. In allogeneic cell therapy the recipient receives cells collected from
a donor. Key issues for a REC to consider include:
 Has the scientific rationale for the cell therapy been explained? (note that cell therapies that
are classed as Advanced Therapy Medicinal Products will undergo rigorous scientific
assessment of the trial application by the MHRA or other regulatory bodies)
 Assessment of efficacy as randomised controlled trial design may not suitable for early trials
especially if invasive procedures are involved eg historical control groups or parallel
observational groups may be used instead
 Have potential risks and benefits been adequately assessed and explained in the participant
information sheet?
 Is the source of the cells documented in the participant information sheet: the patient or a
donor?
 Is the method of delivery ( e.g. intravenous, intramuscular, surgical) explained?
 Is the duration of followup, which may span many years depending on the cell type,
documented in the participant information sheet and included in the consent form?
 If long term immunosuppressant therapy is required is this detailed in the participant
information sheet?
 What quality control and procedures are in place for the cell preparations ( manufacturing,
storage, handling, administration)?
Further sources of information
Cell Therapy Catapult - http://ct.catapult.org.uk/
Science Media Centre Briefing notes on human stem cells – www.sciencemediacentre.org/
EuroStemCell – www.eurostemcell.org/
Lunn JS, Sakowski SA, Hur J, Feldman EL. Stem cell technology for neurodegenerative diseases. Ann
Neurol. 2011 Sep;70(3):353-61. doi:10.1002/ana.22487.