Position statement from the European Society of Gene Therapy (ESGT) Background
News of a serious adverse event in a clinical trial of gene therapy for inherited, X chromosome-linked Severe Combined Immunodeficiency (X-SCID) was made public on October 2, 2002, by the group of French scientists performing the trial, led by Alain Fischer and Marina Cavazzana-Calvo at the Hôpital Necker Enfants Malade in Paris. The investigators announced that one of the patients under treatment had unexpectedly developed a monoclonal lymphoproliferative disease (a leukemialike disorder), and was receiving chemotherapy. ESGT fully supported the investigators' and French regulatory authority's decision to put the trial on hold pending further investigations (see the ESGT statement posted on October 7 on the ESGT web site at http://www.esgt.org). The French investigators decided to share with the scientific community their data on the adverse event by presenting the case at the ESGT annual meeting held in Antibes (France), October 13-16, 2002. Dr Marina Cavazzana-Calvo delivered the presentation on October 16 to an audience of more than 500 scientists and experts in the field, mainly from Europe and the United States.