Lyngstadaas A, Smeland E B
Senter for medisinsk metodevurdering SINTEF Unimed Postboks 124 Blindern 0314 Oslo.
Tidsskr Nor Laegeforen. 2001 Jan 30;121(3):343-8.
This article presents the main conclusions and the recommendations of a multidisciplinary group of experts appointed by the Norwegian Centre for Health Technology Assessment to assess the potential of gene therapy in clinical medicine.
Clinical gene therapy protocols, ongoing or completed with published results, if any, were identified through a systematic survey of descriptive protocols and publications.
So far 3,000-4,000 patients have been treated with gene therapy strategies in more than 400 clinical trials. In Norway the six first patients have been treated with gene therapy at The Norwegian Radium Hospital as part of two approved protocols for treatment of cancer. Gene therapy today is dominated by preclinical and clinical research. Most of the gene therapy protocols identified are in early phases (phases I and II) with few patients in each study; only three of the protocols represent phase III studies. Apart from the use of soluble antisense oligonucleotides against cytomegalovirus, gene therapy is not an established treatment modality today. In early clinical studies, however, promising results have been seen in treatment of cancer, in certain forms of cardiovascular diseases and also in a subgroup of inherited severe combined immunodeficiency.
The expert group recommends that it is now important to build up national competence in the field in two ways: 1) by building up infrastructure in selected milieus; 2) by starting a national programme for gene therapy research including both preclinical and clinical research. The article also considers ethical and legislative aspects and emphasises that gene therapy should continue to be carefully monitored for side effects.
本文介绍了挪威卫生技术评估中心任命的一个多学科专家小组的主要结论和建议，该小组旨在评估基因治疗在临床医学中的潜力。
通过对描述性方案和出版物的系统调查，确定了正在进行或已完成且有发表结果（如有）的临床基因治疗方案。
到目前为止，在400多项临床试验中，已有3000 - 4000名患者接受了基因治疗策略。在挪威，作为两项批准的癌症治疗方案的一部分，挪威镭医院已对首批6名患者进行了基因治疗。如今基因治疗主要由临床前和临床研究主导。所确定的大多数基因治疗方案处于早期阶段（I期和II期），每项研究的患者较少；只有三项方案代表III期研究。除了使用针对巨细胞病毒的可溶性反义寡核苷酸外，基因治疗目前还不是一种既定的治疗方式。然而，在早期临床研究中，在癌症治疗、某些形式的心血管疾病以及遗传性严重联合免疫缺陷的一个亚组中已取得了有前景的结果。
专家组建议，现在通过以下两种方式在该领域建立国家能力很重要：1）在选定环境中建立基础设施；2）启动一项包括临床前和临床研究的国家基因治疗研究计划。本文还考虑了伦理和立法方面，并强调应继续密切监测基因治疗的副作用。
