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[Gene therapy--status and recommendations in Norwegian health care system]
1Senter for medisinsk metodevurdering SINTEF Unimed Postboks 124 Blindern 0314 Oslo. Anita.Lyngstadaas@unimed.sintef.no
Background:
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.
Materials And Methods:
Clinical gene therapy protocols, ongoing or completed with published results, if any, were identified through a systematic survey of descriptive protocols and publications.
Results:
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.
Interpretation:
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.
Insights
Gene therapy, involving over 400 trials, shows promise for cancer and inherited immunodeficiency. National investment in research infrastructure and programs is recommended for its careful clinical development.
Area of Science:
- Biotechnology
- Medical Research
- Genetics
Context:
- Gene therapy is a rapidly evolving field with significant potential in clinical medicine.
- A multidisciplinary expert group was convened to assess its current status and future prospects.
Purpose:
- To evaluate the potential of gene therapy in clinical medicine.
- To provide recommendations for national development and research in gene therapy.
Summary:
- Over 400 clinical trials involving 3,000-4,000 patients have been conducted globally.
- Gene therapy is primarily in early-phase research (I and II), with limited phase III studies.
- Promising results observed in cancer, cardiovascular diseases, and severe combined immunodeficiency, though not yet an established treatment.
Impact:
- Recommends building national competence through infrastructure development and a national research program.
- Highlights the importance of continued monitoring for ethical, legislative, and side effect considerations.