Hovig E, Myklebost O, Aamdal S, Smeland E B
Det Norske Radiumhospital 0310 Oslo.
Tidsskr Nor Laegeforen. 2001 Feb 10;121(4):482-8.
There has been a substantial increase in our understanding of the pathogenesis of cancer over the past decades, and new treatment modalities are being developed on the basis of this knowledge.
The literature is reviewed, and the status of gene therapy protocols approved in Norway is presented.
About 70% of the more than 400 clinical gene therapy studies started are targeted at cancer. Several different principles are used, including gene therapy targeted at tumour suppressor genes, oncogenes, or central signaling molecules, as well as "suicide gene" therapy. In addition, various gene therapy protocols aim at strengthening immune responses. Most studies have been early clinical trials primarily designed to study safety, applicability and toxicity. Several of these phase I and II studies have, however, shown partial remission of tumours and, in rare cases, complete remission, although curation has not yet been shown. In some trials, including TP53 gene therapy trials, effects on tumour size have been observed in up to 50% of the patients. Up until now, only two phase III and one phase II/III studies have been initiated, but results from these studies have not yet been published. The two first gene therapy protocols approved in Norway are also targeted at cancer. So far, six patients in Norway have undergone gene therapy.
As of today, the results should be seen as promising for some of the principles which are being tried out; their clinical importance must, however, be documented in larger controlled clinical trials.
在过去几十年里，我们对癌症发病机制的理解有了大幅提升，基于这些知识，新的治疗方式不断涌现。
回顾相关文献，并介绍挪威批准的基因治疗方案的情况。
已开展的400多项临床基因治疗研究中，约70%针对癌症。采用了多种不同的原理，包括针对肿瘤抑制基因、癌基因或中心信号分子的基因治疗，以及“自杀基因”治疗。此外，各种基因治疗方案旨在增强免疫反应。大多数研究属于早期临床试验，主要目的是研究安全性、适用性和毒性。然而，其中一些I期和II期研究显示肿瘤部分缓解，在极少数情况下完全缓解，尽管尚未证实可治愈。在一些试验中，包括TP53基因治疗试验，高达50%的患者观察到肿瘤大小有变化。到目前为止，仅启动了两项III期和一项II/III期研究，但这些研究结果尚未发表。挪威批准的首批两项基因治疗方案也针对癌症。到目前为止，挪威已有6名患者接受了基因治疗。
截至目前，对于一些正在试验的原理，结果显示颇具前景；然而，其临床重要性必须在更大规模的对照临床试验中得到证实。
