On Monday, 24 January 2005, the French Health Authority AFSSAPS (Agence Francaise des Sécurité Sanitaire des Produits de Santé) reported that a third patient developed a lymphoproliferative disease, out of eleven patients treated in the SCID (Severe Combined Immunodeficiency Disease) gene therapy study conducted by Dr. Alain Fischer at the Hôpital Necker des Enfants Malades, in Paris. This clinical study has therefore been put on hold again. In 2004, after an updated benefit/risk analysis, the principal investigator, Dr. Alain Fischer, continued this SCID gene therapy study trial after it had been put on hold due to two cases of leukaemia which occured in late 2002. The two affected children had been treated at the age of three and six months, respectively. The third and most recent case of a lymphoproliferative disease, possibly comparable to the two previous leukaemias, is a child whose life-threatening congenital immunodeficiency disease - SCID-X1 - had been treated at the age of nine months.
"As in the first two cases of leukaemia, not only the Paul-Ehrlich-Institut, but also the Commission of Somatic Gene Therapy (
Kommission Somatische Gentherapie) of the Scientific Council of the German Medical Association (Bundesärztekammer) and the Gene Therapy Expert Group (GTEG) at the European Medicines Agency (EMEA) will analyse the background of this new case", explained Prof. Klaus Cichutek, Vice President of the Paul-Ehrlich-Institut and chairman of EMEA - GTEG in London. In his view, there is no need for immediate action in Germany, since according to current knowledge, as he explained, the causes for leukaemia development assumed by the experts, are not present in the gene therapy studies performed in Germany. "However, the exact outcome of the analysis of the causes underlying disease have to be awaited. Measures would have to be taken if, on the basis of clinical trials or animal experiments, we also found evidence of oncogenesis after the application of replication-defective retroviral vectors in types of gene therapy clinical studies other than SCID-X1 gene therapy"
, said Prof. Cichutek.
In autumn 2004, on the occasion of the International Conference of the European Society of Gene Therapy (ESGT), Dr. Fischer had reported that one of the two children who had developed leukaemia had died in the meantime.
A comparable SCID gene therapy study has been performed by Dr. Thrasher in London for some years. This study, in which up to now not a single case of leukaemia has occurred, has so far not been put on hold.
The German Registry for Somatic Gene Transfer Studies ("Deutsches Register somatische Gentransferstudien" (DeReG), provides information on gene therapy clinical studies currently on-going in Germany.
If you need additional information, please contact Dr. Susanne Stöcker.
Updated: 28.01.2005
