Activartis Receives Orphan Drug Designation for its Cancer Immune Therapy AV0113
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|Thursday, 07 March 2013 15:45 (UTC + 1)|
Vienna, Austria, March 7, 2013 / B3C newswire / - The European Medicines Agency recently awarded the Austrian biotech company Activartis 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
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.
EMA: First proof of efficacy expected
The director of the team that developed this new form of Cancer Immune Therapy, Thomas Felzmann, who is now CEO of Activartis, says: “Acquiring orphan drug designation is an important milestone for us and a strong signal to others. We are convinced that this approach has great potential and now an independent party, the European Medicines Agency, has confirmed this. However, it will still take another year or so before robust evidence for a benefit of AV0113 for patient’s suffering from Glioblastoma will become available.”
The majority of neuro-surgical/neuro-oncological clinics in Austria currently participate in a randomised phase II study, GBM-Vax, designed to deliver evidence for AV0113’s efficacy. GBM-Vax has recruited close to 100 Glioblastoma patients. Now we have to wait and see whether patients survive longer when treated with the AV0113 Cancer Immune Therapy.
Keyword: Orphan Drug Designation