SAFETY WITHIN GIANT

Public acceptance is key to new techniques such as gene therapy. The EU Clinical Trials Directive requires the manufacturing of all Investigational Medicinal Products (IMPs) to the standards of Good Manufacturing Practice (GMP). The project consortium includes a GMP vector production facility and clinical facilities with scientific and ethical permission to carry out human cytotoxic gene therapy trials and aims to set up Phase I clinical trials in four countries: France, the Netherlands, Sweden and the UK. Phase I trials are aimed at demonstrating the safety, or non-toxicity, of a therapy, but in some cases they can also give indications of effectiveness in treating the disease. There are four steps or phases before a therapy can be used widely. “By combining four Phase I trials at once” says Professor Maitland, “GIANT researchers hope to have results of statistical significance, giving some indications before going to the Phase II stage of testing effectiveness through placebo controls.”

While second and third-generation vectors are under investigation by the project, the clinical trials will only be on older, more established first-generation vectors. The GIANT participants have a long record of EU-based scientific collaboration and expertise in generating ethically approved clinical vectors. A Scientific Advisory Board of external experts has also been recruited to the project to advise on clinical trials and ethical considerations.