The European Medicines Agency (EMA) has granted Orphan Drug Designation for Activision Artis' novel cancer immunotherapy. Specifically refers to the orphan drug designation for the application of Activision Artis' AV0113-drug in the treatment of glioma, a type of brain tumor that the EU meets about one in 10,000 people.

As orphan diseases - rare diseases are known, there is little for the research and development interests and for which therefore no adequate treatments available.

Basically Activision Artis' AV0113 technology can be used against all cancers. The technology is based on a therapeutic in clinical development, new, patented strategy of tumor vaccination with dendritic cells. The dendritic cells are central regulatory elements of the immune system. The aim is to enable the immune system in a position to provide the cancer under the control of the immune system.

The mechanism of action of the active immunotherapy of cancer AV0113-Activ Artis is the mobilization of the immune system of the patient/ inside. Tumour cells are the basis of their antigens (determinants of tumor cells differ from normal cells) are recognised and destroyed. The effect of therapy makes itself elements and mechanisms of the immune system uses to advantage and where this fails in the defense against cancer. As autologous tissue tumor cells by the immune system is not typically considered to be hazardous. The Activ Artis therapy "deceptive" Dendritic cells to do the right thing, namely the tumor as a threat to exercise and get an immune reaction.

The dendritic cells of the cancer patient/ inside are loaded with tumor antigens from their own cancer tissue. These antigens are processed by the dendritic cell and transported to the cell membrane, where they are available for presentation to T cells. To induce an immune response that was not enough, however. The above-mentioned illusion of dendritic cells is that they are brought into contact with microbial danger signals. Certain molecules which are present in micro-organisms, but not in higher organisms signalize the dendritic cell in their environment that microorganisms are penetrated and thus pose a threat to the organism.

Tumour cells arise from normal cells in the body the cancer patient/ internally and therefore do not have danger signals for recognition by dendritic cells. The critical and unique part of Activision Artis' AV0113 technology is that loaded with tumor antigens, dendritic cells are brought into contact with a danger signal: lipopolysaccharides bacterial endotoxins. This causes the dendritic cell to accept a potent pro-inflammatory and immune-function mode. After returning of dendritic cells in the body of the / patient / to activate tumor-antigen specific T cells, most importantly including the cytotoxic (cell-killing) T cells that are able to recognize cancer cells and destroy them.

To give an Orphan Drug designation, EMA expects the first evidence for a possible efficacy of the drug - but still no definitive proof. For patient/ interior with a particularly aggressive form of glioma, the glioblastoma multiforme, such references have now been documented by Activision Artis. Worldwide each year 50,000 people are affected by a new disease with this aggressive type of brain tumour. With an average of patient/ inside with a glioblastoma little more than a year. In the five-year period, over 90 per cent of the dying patient/ inside. The classic forms of cancer treatment - surgery, radiation and chemotherapy - put the patient/ inside only a slight prolongation of life by a few months.

The leader of the team that developed this new form of cancer immunotherapy, Felzmann Thomas, who is now CEO of Activision Artis explained, "The orphan drug designation is an important milestone for us and a strong signal. We believe that this approach has great potential. Now we have to confirm independently, the European Medicines Agency. It will still take about a year until we have robust evidence that patients suffering from glioblastoma/ inside actually have a survival advantage.”

Most neurosurgery/ neuro-oncology clinics in Austria are currently taking part in a randomized phase II study "GBM-Vax" whose design is designed to show evidence for the effectiveness of AV0113. GBM-Vax has been included nearly 100 patient/ inside with glioblastoma. Now we have to wait and observe whether the patient is treated with the cancer immunotherapy/ inside have a survival advantage.

Diseases that are neglected because of their rarity of biomedical research and development often are referred to as orphan diseases, orphan diseases, ie diseases about which nobody cares. A so-called orphan drug designation is a medical drug by European and American Food and Drug Administration, FDA and EMA, then awarded if so one of these very rare diseases can be treated. If an orphan drug designation to be granted, the health authorities check next to the actual rarity of the disease and whether the developer of the drug may show the first evidence of possible efficacy. Benefits of orphan drug designation are accelerated approval and market exclusivity in Europe for 10 years from approval in the US for 7 years, where the US Food and Drug Administration granted orphan drug designation also a. An orphan drug designation to fostering the development of drugs for people suffering from a rare, life-threatening diseases.