Gene Therapy

Data and Facts
Many diseases cannot be cured with the typical medical methods available today. Great hope exists that it will be possible in the future to provide long-term treatment for inherited disease as well as chronic diseases and some forms of cancer with gene therapy. Gene therapy involves placing curative genes into human body cells. To do this, one needs a suitable vehicle, referred to as a vector. "Empty" and therefore harmless viruses are frequently used for this. These bind to specific cells of the body and inject the "effective" genetic information. In general, one differentiates between altering the genetic material of body cells (somatic gene therapy) and of germinal cells. With somatic gene therapy, the genetic modifications are not passed to the next generation because only selected body cells and no germinal cells are altered. One also differentiates between in-vivo and ex-vivo therapies. With in vivo treatment, the intact gene is inserted directly into the diseased area (e.g. an organ, the circulatory system or a muscle) inside the selected vehicle. With ex-vivo gene therapy, specific cells are first removed from the patient, genetically modified in the laboratory, cultivated and reinjected into the patient's body. Initially, gene therapy will be able to treat so-called monogenetic diseases, in other words, diseases that are caused by a single defective gene (e.g. hemophilia). Nevertheless, approximately 70 percent of the gene therapy studies underway today involve forms of cancer for which multiple defective genes are often present.

What Does the BMBF Do?
Since 1995, the BMBF has supported various focus areas that are concerned exclusively with the development of new methods for somatic gene therapy. Approximately 45 million EURO is used to fund over 165 projects in which new therapy approaches for diverse diseases are being investigated, generally within joint research consortia. In addition to the actual gene transfer into human cells, the research projects are also involved in the establishment of disease-specific animal models. Only in this way can it be investigated whether promising therapies can also be transferred to a living organism. Only after such tests can the procedure also be applied to humans. Another goal of the funding measure is to improve cooperation between theoretical and clinical workgroups and to stimulate increased integration of industrial partners. To date, 45 patent applications have resulted from the projects, a series of associated biotech companies have been founded and industrial partners have already been integrated in over 5 percent of the projects. Due to the continuing research needs and the foreseeable therapy possibilities, the BMBF will continue the funding beyond the year 2003.