Clinical gene therapy - first international conference. 24-26 January 2002, Groningen, the Netherlands

• Published in: IDrugs : the investigational drugs journal Vol. 5; no. 3; p. 209
• Main Author: Vandendriessche, Thiery C
• Format: Journal Article
• Language: English
• Published: England 01.03.2002
• ISSN: 1369-7056

The first International Conference on Clinical Gene Therapy focused primarily on gene therapy for cardiovascular disorders, genetic diseases (hemophilia, cystic fibrosis) and cancer, with particular emphasis on clinical trials and advanced preclinical studies. Recent improvements in vector technologies led to the first demonstration that gene therapy could cure a disease in a clinically relevant animal model of hemophilia. This significant progress at the preclinical level, using both viral and non-viral vectors, paved the way for phase I clinical trials in patients suffering from hemophilia A. The first report on hemophilia A gene therapy using a non-viral ex vivo approach was encouraging and revealed modest improvements in clinical endpoints. The status of clinical gene therapy for the treatment of peripheral limb and myocardial ischemia by therapeutic angiogenesis was highlighted as showing an apparent increase in collateral development and reduced limb or myocardial ischemia in some patients. Gene therapy for cystic fibrosis turned out to be a much tougher nut to crack than initially assumed, and the hopes for, and hurdles faced by, gene therapy approaches for this disease were discussed. Phase III clinical trials using retroviral-based suicide gene therapy for cancer revealed no significant therapeutic benefit in patients suffering from glioblastoma multiforme. Nevertheless, new and promising approaches for cancer gene therapy are being developed that rely on the use of targetable vectors and conditionally replicating vectors that replicate specifically in cancer cells; this may overcome some of the bottlenecks of cancer gene therapy by improving therapeutic efficacy. Although few diseases have been treated effectively by gene therapy so far, the stage appears set for new and significant advances in clinical trials.