The European Medicines Agency recently awarded the Austrian biotech company ctivartis an Orphan Drug Designation for its innovative Cancer Immune Therapy AV0113. The Orphan Drug Designation applies specifically to the use of AV0113 for the treatment of glioma, a type of brain tumour, which afflicts around one in 10.000 people in the EU. The term Orphan Disease refers to rare diseases which do not attract much interest in the way of research and development and thus for which there are no appropriate treatments available.

         Activartis’ AV0113 Cancer Immune Therapy can basically be used to fight every type of cancer. The therapeutic technology is based on a patented procedure in which a cancer patient’s immune system is primed to fight the tumour and eventually control its growth. This is based on the use of Dendritic Cells, the key regulatory elements of the immune system, that are just as the tumour tissue derived from the patient.

         Active cancer immune therapy based on Dendritic Cells
AV0113 activates the patient’s immune system, with tumour cells being identified on the basis of their antigens and destroyed. The therapy makes use of elements and mechanisms of the immune system and gets to work where these fail. As tumour cells are the body’s own tissue, the immune system normally doesn’t identify them as dangerous. Activartis’ AV0113 Cancer Immune Therapy “tricks” Dendritic Cells, and onsequently a cancer patient’s immune system, into doing the right thing, i.e. to perceive the tumour as a threat and to trigger adequate defence mechanisms.

        The Dendritic Cells are charged with tumour-derived antigens, determinants that distinguish a tumour cell from a normal cell. These antigens are processed by the Dendritic Cell and shuttled to the cell surface in order to present them to T-cells. This, however, is not sufficient to prime an immune response against the tumour antigens. The “trick” referred to above is contacting Dendritic Cells with a microbial danger signal. Certain molecules, that are present in microorganisms but not in higher organisms signal the Dendritic Cell the presence of a microorganism in its surroundings and hence danger to the organism.

        As tumour cells originate from a cancer patient’s normal cells, they do not provide such danger molecules for recognition by the Dendritic Cell. The critical and unique part of Activartis’ AV0113 technology is exposing tumour antigen-charged Dendritic Cells to one of these danger molecules: Lipopolysaccharides, the bacterial endotoxins. This causes the Dendritic Cell to assume a potently immune stimulatory and
pro-inflammatory mode of action. Upon returning these Dendritic Cells to the patient they activate tumour-specific T-cells, most importantly the so-called Cytotoxic T-cells, which become enabled to recognise and destroy tumour cells.