New gene therapy trial for X-linked CGD gets go-ahead

The British Medicine and Healthcare Regulatory Agency (MHRA) has approved a new Phase I / II gene therapy clinical trial for X-linked CGD to start at Great Ormond Street Hospital in London. This will form part of a multicentre trial with centres in Germany, Switzerland, France and US involved. In total the European trial will include 20 patients, 5 per site to be treated who will be followed up for 2 years.

The trial uses the lentiviral vector designed and developed through CGD Society funding of over £1M to Professor Adrian Thrasher at Great Ormond Hospital, London and Dr Manuel Grez at Georg Speyer Haus in Frankfurt. Further modifications and clinical production of the vector so it could be brought into the clinic were made possible by funding from Généthon, a not-for-profit research centre in France and from the European Commission.

The trials will be headed by Professor Adrian Thrasher and Bobby Gaspar at Great Ormond Street Hospital in London, in Germany by Professor Hubert Serve at the University Hospital in Frankfurt, in Switzerland by Professors Reinhard Seger and Janine Reichenbach at Children's Hospital of Zurich and in France by Professors Alain Fischer, Marina Cavazzana-Calvo, Stéphane Blanche and Salima Hacein-Bey-Abina at the Necker Hospital for Sick Children in Paris.

This multicenter international effort, called Net4CGD, Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) is a large-scale integrating project in Health Research of the European Commission 7th framework programme (FP7). Its focus is on the clinical development of gene therapy so that it can rapidly become a new treatment option for patients with X-linked CGD. The Net4CGD consortium consists of 11 partners Institutions and is coordinated by Genethon.

‘Although there has been good progress in BMT for CGD but we know this approach is not possible for all patients. For those X-linked CGD patients who may not be good candidates for BMT, gene therapy represents an important new treatment option. We learned a lot over the last few years in terms of making gene therapy more effective and also safer, so now is a good time to conduct trials using new technologies in patients with CGD. The progress made would not have been possible without the research investment made by the CGD Society over the last 15 years which has allowed other more wealthy funding bodies to build upon bringing this next generation vector to clinic’, said Professor Adrian Thrasher.