Opinion paper on the current status of the regulation of gene therapy in Europe

The potential applications of gene therapy are many, extending from monogenic hereditary diseases to acquired and multifactorial disorders. Therapeutic gene transfer addressing such a variety of conditions is currently being investigated, creating therapeutic options for diseases where none had previously been available. The field of gene therapy represents one of the most challenging therapeutics for the new millennium. As such the concept might have been oversold ahead of its time. Nevertheless, is some instances, potential clinical efficacy is currently being found with reports of significant successes, for example, in inherited severe combined immunodeficiency disorders (SCID), hemophilia, arteritis obliterans, and even cancer. The development of recombinant DNA technology has caused fear in the public and speculation regarding its potential risks. Public reaction to the implementation of gene therapy is at best ambivalent, ranging from enthusiasm about its formidable therapeutic promises to apprehension concerning its putative harmful consequences. Indeed, the report of accidents in the United States resulted in a broad debate on the subject of gene therapy regulation brought to the attention of the U.S. Senate and Government. In addition, the recent occurrence of a malignant proliferation of the hematopoietic system in one gene therapy-treated SCID patient has been reported (www.astg.org). with a monoclonal insertion of the retrovirus vector at a sensitive locus (LMO2).