Project "Gene Therapy"
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National Program of research and development

by V. Vonka (Institute of Haematology and Blood Transfusion), Prague, May 2002,
synopsis by T. Adamec for the web site of the Biotrin Association

It is possible to cover all the procedures that use transfer of genetic material into cells of a patient for therapeutic purposes with the term gene therapy (GT). Theoretically GT methodology is applicable whenever molecular nature of a disease is known. As information on molecular mechanism of pathogenesis increases, also the scope of GT indication grows.

At present, GT fights many problems, but few doubt its implementation in human medicine is inevitable and in the immediate years, it will principally change the way many human diseases are cured.

Although, when GT was developed it was mainly meant to cure inherited diseases, most clinical studies are focused in these days on the acquired diseases. Among those, tumours are dominating but GT procedures are intensively explored in therapy of cardiovascular and "metabolic diseases, degenerative neural diseases, AIDS, transplantation medicine and some other areas".

Present problems of gene therapy can be divided into three categories:

  1. Technical problems concern mainly with the vectors of genetic material, which are of viral origin so far. They must fulfil many demands regarding effectiveness and safety especially. Vectors, which would meet all the demands completely, are not available yet.
  2. Methodological problems in searching for optimum strategies for reaching of the desired effect. Proper strategy undoubtedly depends on a type of disease, its clinical stage and on many other circumstances. As we gather new pieces of knowledge, individual procedures are modified and revised.
  3. Ethic problems. GT risks cannot be overlooked. Each proposal of clinic study must be evaluated from the view of the law in force and must be in accordance with the latest scientific findings. Patient must be informed about both possible benefits and risks. The latter should be carefully considered especially in cases when an effective alternative therapy exists.

Present situation in the Czech Republic

Several local institutions and laboratories deal with GT for some years.(*) Although, these workplaces have an ongoing support of grant agencies and constantly reach results on good international level, present status of GT in the Czech Republic cannot be considered satisfactory. Not one out of several hundred clinical studies in the GT area that are going on, is performed here. The unfavourable situation has several reasons that are closely related. They are:

  1. until recent, there is only a little interest of our clinicians,
  2. missing legislation that would set rules for GT clinical studies,
  3. lack of an establishment that would be able to prepare experimental bio-preparations in accordance with the Good Manufacturing Practice,
  4. the State Institute for Drug Control (SÚKL) does not have any group at disposal that would be able to control new preparations competently, 
  5. low appreciation of our public on GT. Consequently, there is little public pressure to implement and examine GT here.

Measures for development of GT in the CR

The goal of the proposed project is to create conditions for stimulation of research of GT and for the start of clinical studies using GT here. Thus our country would join the most developed countries when implementing new biotechnology. The task includes several components:

  1. Scientific-research program. Regarding the present state the development of therapeutic genetic anti-tumour vaccines should have the priority, as there is hope to begin clinical studies in the horizon of several years. Simultaneously, pre-clinical research of GT of cardiovascular and some acquired metabolic diseases should be started and supported. And also, any well-founded initiative in other areas should be supported.
  2. Legal framework for GT. It is necessary to have a law in the shortest possible time, which would enable clinic use of GT in the CR. A committee established with the Second Medical Faculty of the Charles University. should provide professional ground for the law. Legal frame of regulation should be based on similar laws, which have been adopted in the EU countries.
  3. Establishment of a modern production place. It is necessary to establish a workplace that would be able to make preparations for GT clinical studies in accordance with the Good Manufacturing Practice. The First Medical Faculty of the Charles University should seek accreditation to provide such a workplace; the Ministry of Agriculture and the Academy of Science and probably pharmaceutical companies should share funding.
  4. Establishment of biological control centre. Within the State Institute for Drug Control (SÚKL) it is necessary to form a work group that would be able to control new preparations for clinic tests in a qualified way, i.e. in accordance with the latest findings of medical sciences.
  5. Education and information for the public. GT should be made part of curriculum at medical, pharmaceutical and veterinary faculties. It is also necessary to inform the public about existence, benefits and risks of GT. Information events should be organised so that they attract attention of greatest possible part of population. The Czech Medical Society of J.E.Purkyně should play an important role in these efforts.

(*) Department of Tumour Immunology ÚMG AV CR, Praha, Department of Experimental Virology UHKT, Praha, Department of Cellular Biology and Physiology UHKT, Praha, Institute of Medical Biology and Genetics LF UK, Hradec Kralove and Research Institute of Veterinary Medicine, Brno etc. 

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