CLINICAL TRIAL OF GENE THERAPY FOR WISKOTT-ALDRICH SYNDROME

Wiskott-Aldrich Syndrome (WAS) is a rare genetic disease in which the functions of the immune cells present in the blood are compromised. Patients affected by WAS have a reduced life expectancy due to reduced capacity to control infections, occurrence of severe bleedings and susceptibility to develop cancer. To date, the majority of affected children have no curative therapeutic options available. We propose to implement a gene therapy clinical trial for WAS based on our preclinical studies and on the successful treatment of children with ADA-SCID using a similar approach at our center. It will consist in the repair of the blood cell progenitors that will be isolated from the bone marrow of patients. A healthy copy of the gene responsible for the disease will be transferred into these progenitors ex vivo via a viral vector before re-infusion. The aim of this clinical research protocol is to demonstrate the safety and efficacy of this experimental treatment. We will provide the care to patients undergoing gene therapy in the dedicated Pediatric Clinical Research Unit. A similar clinical trial will be conducted at the time in London by our collaborator A. Thrasher and colleagues in order to reach more rapidly conclusions about the outcome of the same treatment. It is expected that this trial will provide clinical benefit for WAS patients and will contribute to widening the field of application of gene therapy.