In the fight against common diseases such as cancer the strategy is clear: scientists are working flat out on research, methods of early detection and treatments. That the demand is enormous is not surprising: according to the Federal Statistics Office, around 26 percent of all deaths in Germany in 2011 were due to cancer. So it is no wonder that patients, researchers and pharmaceutical companies want to see effective drugs developed fast.
But what is the situation with drugs to treat diseases which affect only a few people? “These drugs are often neglected by pharmaceutical companies because they are economically unattractive,” says Dr. Karin Aufenvenne, a biologist, who personally experienced the problem over the past few years. Around one year ago she and Prof. Heiko Traupe, senior physician for dermatology at the skin clinic at Münster University Hospital, achieved a breakthrough in treating a special form of ichthyosis with medication. This highly unpleasant skin disease, often known as fish-scale disease, is caused by genetic defects and occurs directly after birth. Patients suffer from an extreme thickening of the top layer of the skin, which manifests itself in a dark brown, lamellar desquamation covering the entire body. “Around 2,500 people in Europe are estimated to have the disease – which means it is ultra-rare,” says Karin Aufenvenne.For this reason no pharmaceutical company could be found which was prepared to progress and - above all - market the therapy developed at Münster University (WWU) in close cooperation with the working group headed by Dr. Margitta Dathe at the Leibniz Institute of Molecular Pharmacology (FMP) in Berlin. This situation could, however, change shortly. The reason is that the medication was recently awarded orphan drug status by the European Union – an unprecedented event at WWU and FMP. With the official EU designation as a “drug to treat rare afflictions”, it becomes more economically attractive. “Pharmaceutical companies that buy the rights to such a drug do not have to pay any approval costs, for example,” explains Karin Aufenvenne. “And after regulatory approval they have exclusive rights to the drug for ten years.” In Münster there is a strong desire to find a company attracted by these incentives. “The distress felt by patients who have this disease is enormous – not only due to the physical symptoms, but also because of the mental suffering.”
Dr. Christina Heimken | idw
Electrical 'switch' in brain's capillary network monitors activity and controls blood flow
27.03.2017 | Larner College of Medicine at the University of Vermont
Laser activated gold pyramids could deliver drugs, DNA into cells without harm
24.03.2017 | Harvard John A. Paulson School of Engineering and Applied Sciences
Astronomers from Bonn and Tautenburg in Thuringia (Germany) used the 100-m radio telescope at Effelsberg to observe several galaxy clusters. At the edges of these large accumulations of dark matter, stellar systems (galaxies), hot gas, and charged particles, they found magnetic fields that are exceptionally ordered over distances of many million light years. This makes them the most extended magnetic fields in the universe known so far.
The results will be published on March 22 in the journal „Astronomy & Astrophysics“.
Galaxy clusters are the largest gravitationally bound structures in the universe. With a typical extent of about 10 million light years, i.e. 100 times the...
Researchers at the Goethe University Frankfurt, together with partners from the University of Tübingen in Germany and Queen Mary University as well as Francis Crick Institute from London (UK) have developed a novel technology to decipher the secret ubiquitin code.
Ubiquitin is a small protein that can be linked to other cellular proteins, thereby controlling and modulating their functions. The attachment occurs in many...
In the eternal search for next generation high-efficiency solar cells and LEDs, scientists at Los Alamos National Laboratory and their partners are creating...
Silicon nanosheets are thin, two-dimensional layers with exceptional optoelectronic properties very similar to those of graphene. Albeit, the nanosheets are less stable. Now researchers at the Technical University of Munich (TUM) have, for the first time ever, produced a composite material combining silicon nanosheets and a polymer that is both UV-resistant and easy to process. This brings the scientists a significant step closer to industrial applications like flexible displays and photosensors.
Silicon nanosheets are thin, two-dimensional layers with exceptional optoelectronic properties very similar to those of graphene. Albeit, the nanosheets are...
Enzymes behave differently in a test tube compared with the molecular scrum of a living cell. Chemists from the University of Basel have now been able to simulate these confined natural conditions in artificial vesicles for the first time. As reported in the academic journal Small, the results are offering better insight into the development of nanoreactors and artificial organelles.
Enzymes behave differently in a test tube compared with the molecular scrum of a living cell. Chemists from the University of Basel have now been able to...
20.03.2017 | Event News
14.03.2017 | Event News
07.03.2017 | Event News
27.03.2017 | Earth Sciences
27.03.2017 | Life Sciences
27.03.2017 | Life Sciences