MEETING REVIEW:

Genome Medicine:
Gene Therapy for the Millennium

Dr Anna Rüdeberg

My participation in the meeting on Gene Therapy was not only motivated by the highly appreciated invitation by Barbara Palys and Dieter Gruenert, but also by my professional interest in CF and other genetic disorders. The attractiveness of this meeting for me was much increased by the fact that it was held in Rome, the city were I was born and which I left 30 years ago. In the heart of Rome, 50 meters from the Spanish Steps, in a congress center where I used to meet school friends, I felt at home more than ever: at home in Rome, but also at home in these specific areas of medicine and science.

My attention was focused particularly on the current knowledge of gene therapy, on its ethical implications and on the socio-political issues. These areas were treated in sessions where research workers, medical personnel, patients, members of family support groups, and socio-political professionals were given the opportunity to exchange viewpoints.

"…it is still wholly experimental."

Before delving into the complexity of the ethical implications, we were brought up to date in gene discovery, on new animal models and on the tools for evaluation of disease progression and therapy. One of the most interesting discussions arose on the fetal somatic gene therapy, also in my eyes a concrete and eventually possible way of therapy. Charles Coutelle, UK, made it clear to us all, however, that it is still wholly experimental. The conflicting reactions and opinions in the field of gene therapy are probably a consequence of ignorance, confusion and misunderstanding. Proper information and the decrease of economic, political and journalistic manipulations will work out in favor of the patients concerned.

"…often give the impression that these hypotheses are already proven."

I am surprised by the discrepancy between the rationality of the scientific efforts to improve therapy and the ethical issues involved. Although the therapeutic methodologies for cure and prevention of genetic diseases are still based on theoretical and experimental hypotheses, the media (especially press and television) often give the impression that these hypotheses are already proven. This has ethical implications, which, although the methods would be truly welcome, may create problematic issues due to lack of correct information. Thus, erroneous information has already generated ludicrous accusations, such as "playing God", deliberate manipulation of the germ-line and the creation of "designer babies". The more people will be informed, the better they will understand the ongoing research and the more transparent will be the final outcome.

The complexity of the scientific hypotheses is vast, the theoretical possibilities are of an unlimited number, but the solutions put into practice are relatively scarce and often very astute and tricky. The methods finally used must not only be effective, but they must also be safe.
According to several speakers, at present fetal somatic gene therapy is also a purely experimental approach, using animal models to assess the choice of vectors, route of administration and candidate diseases. It is self-evident that the various animal models have to be tested for their true relevance to each specific illness.

The experimental gene therapy strategy requires more exploration in some areas, among these reasons for, and risks involved, in prenatal gene therapy; the possibility of inadvertent modification of the germ-line and, of course, all the particular prerequisites which have to be considered, before application in humans can be carried out.

Dorothy C. Werts, U.S.A. described the influence of the Ethics Committee of HUGO (the Human Genome Organization) in the field of gene therapy. This committee has developed a statement to curb public fears. The statement supports research on somatic cell therapy, urges the establishment of national ethics committees (where such do not already exist) that include gene therapy in their mandates, recognizes the need for ongoing oversight, requires disclosure of researcher conflicts of interest to patients, urges establishment of schemes of indemnification for those harmed by gene therapy research, and proposes discussion of possible future uses of gene therapy for enhancement and germ-line interventions.

"In depth information about benefits and risks should be provided…"

Once the safety and reliability of a gene therapeutic method has been proven, the time will have come to address other questions regarding this specific method with the help of social and political authorities of the countries concerned. These questions comprise cost/effectiveness calculations, equal accessibility, and genetic as well as prenatal screening. To become truly preventive, gene therapy will require universal medical support and broad ethical acceptance. In depth information about benefits and risks should be provided, not only to the families with genetic diseases and the health professionals working with them, but also to the lay public.

"…the journey to reach the goal will be full of obstacles."

I left the congress with a deep conviction that the presentations and the discussions had produced confidence in the future development of this special kind of therapy, in which the integrity of the genome will be protected and safe integrating vectors introduced. The immense research efforts in developing gene therapies are accompanied by similar socio-political efforts to respect and protect the rights of all parties concerned. These efforts inspire a well-founded hope, even if we know that the time needed will be long and the journey to reach the goal will be full of obstacles.

Anna Rüdeberg,
IACFA Medical advisor