Authors' objectives:

To summarise the available evidence on the current status and use of gene therapy.

Authors' recommendations: 1. Three Norwegian patients have been treated with gene therapy against cancer. Gene therapy is today, with a minor exception (treatment of a special viral eye infection), not an established treatment modality in Norway. Norwegian patients should only be offered gene therapy as part of clinical studies. 2. Gene therapy has developed to a point where it is important to build up national competence in the field. The Norwegian Ministry of Health and Social Affairs has recommended that government funds be directed towards competence building at two centres in Norway, Haukeland Hospital and The Norwegian Radium Hospital. The expert group thoroughly discussed a national gene therapy effort in a 5 year perspective. It was agreed that two different strategies should be used: a.To build up infrastructure at certain selected milieus. b.To start a national program for gene therapy research, which should include both preclinical and clinical research. When gene therapy eventually becomes an established treatment modality, it will be necessary to establish facilities at the different regional hospitals and at The Norwegian Radium Hospital. It is suggested that the Ministry establish a scientific steering committee for the evaluation of projects that will be generated from these different centres. 3. Ethical considerations The expert group considers somatic gene therapy to be ethically sound and finds that this does not represent a new ethical principle. The expert group confirms that gene therapy, for the time being, must be reserved for treatment of serious disease. It is important to point out that both children and adults should be evaluated for gene therapy research. Gene therapy research should not be avoided among in children just because the subjects are children. The expert group finds that gene therapy in utero should not be performed until more knowledge is available. Germline gene therapy or gene manipulation of embryos or germ cells should be forbidden in accordance with international consensus and Norwegian law. Genetic enhancement should not be allowed under any circumstances. It is important that the ethical discussions concerning gene therapy be on-going, in particular with respect to new gene therapy protocols. Research on the ethical issues related to gene therapy should be supported. The expert group maintains the necessity for a continued and informed public debate regarding these issues, in addition to the formal regulations and routines for approval which today regulate human gene therapy. 4. The expert group states that it is important that the safety and side effects of gene therapy continue to be thoroughly investigated and supervised. 5. The expert group considers it important to obtain more convenient procedures regarding the approval of gene therapy studies while at the same time ensuring that the process still covers the needs of society with respect to control and ethical considerations. It is suggested that three applications should be sufficient, one for laboratory approval, one for the ethical committee and yet another for approval of drug testing. It is recommended that the Norwegian Medicines Control Authority be augmented with a specific advisory committee for gene therapy, similar to the current situation in England. 6. The different procedures on gene therapy should be regulated with different safety restrictions. Thus, treatment with ribozymes and antisense oligonucleotides in soluble form, which is reminiscent of conventional drug treatment, should have more simplified safety demands than other gene therapy methods. It is recommended that smart viruses and gene marking studies, which are not included in the definition of the gene therapy in this report, should be regulated like gene therapy. 7. Gene therapy is still in an early phase of clinical research and it is difficult to foresee the influence of gene therapy in the Norwegian health care system in the future. As the results of several phase III clinical studies will be available in the next couple of years, it is recommended that a new evaluation of this field be performed within three to five years. This includes updating of the gene therapy databases. 8. A national strategy and development of the gene therapy field is important to achieve. This will make it possible to systematically prioritise different gene therapy methods, to build up national competence in the field and to establish gene therapy as regular treatment whenever this will be indicated. A strengthening of the transfer between basic and clinical research (translational research) within gene therapy will prevent unnecessary pressure for establishment of such new and exciting methods before their clinical effect has been demonstrated, and thus also prevent the inappropriate and uncoordinated establishment of new methods at the national level.

Authors' methods: Overview

Project Status: Completed

URL for project: http://www.nokc.no/

Year Published: 2000

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Norway

Organisation Name: Norwegian Institute of Public Health

Contact Address: Universitetsgata 2, Postbox 7004 St. Olavs plass, NO-0310 Oslo NORWAY. Tel: +47 23 25 50 00; Fax: +47 23 25 50 10;

Contact Name: Berit.Morland@nokc.no, dagny.fredheim@nokc.no

Contact Email: Berit.Morland@nokc.no, dagny.fredheim@nokc.no

Copyright: The Norwegian Knowledge Centre for the Health Services