Severe immune deficiency trial approved

A clinical trial of a gene therapy for the rare immunodeficiency, Wiskott-Aldrich syndrome (WAS), has been approved in France and the UK. It will be sponsored by Généthon, the not-for-profit biotherapy laboratory operated by the French Muscular Dystrophy Association (AFM), with funding from France's annual Telethon. 

The French and British health authorities have authorised the phase I/II trial, which will be conducted both at Necker Children's Hospital, Paris, and at London's Great Ormond Street Hospital.   

WAS is a severe, X-linked immunodeficiency which results in haemorrhage, recurrent infection and eczema. It is almost always fatal in affected children. Although a bone marrow transplant can re-establish the patient's immune system, this requires a compatible donor and carries a risk of failure. Gene therapy is a major source of hope, therefore.   

This trial is the result of research initiated in 2002 in the Généthon laboratory. The researchers have developed an ex-vivo approach that uses an HIV-derived lentiviral vector to transfer genes into autologous CD34+ haematopoietic stem cells from the patient.  

"This trial is enabling the Généthon laboratory to demonstrate its world-class expertise in the production of gene-based medicines and the preparation and implementation of clinical trials." stated AFM and Généthon president, Laurence Tiennot-Herment. "It confirms Généthon's ability to become a major international player in demonstrating the efficacy of gene therapy for rare diseases."  

Généthon is a leading centre for preclinical and clinical research and development in the field of gene therapy. It has a biomanufacturing platform for clinical-grade vectors and is involved in building the world's largest facility for pre-industrial pilot production at Evry, France, that is scheduled to open in early 2011.