Scientists at IMBA – Institute of Molecular Biotechnology of the Austrian Academy of Sciences in Vienna have made a major advancement towards a future therapy for butterfly children. A treatment with fibroblasts generated from induced pluripotent stem cells has been highly successful in mice. The next step is to establish this method in humans.
“Butterfly children” suffer from Epidermolysis Bullosa (EB), a debilitating skin disease. It is caused by a genetic defect that leads to a deficiency or complete lack of various structural proteins. In one particularly severe form, the protein collagen 7 is either missing or present only in insufficient amounts.
If that bond is missing, the skin forms blisters or tears at the slightest mechanical pressure, leading to wounds and inflammation that require extensive treatment with creams and bandages. Often these constant lesions also lead to aggressive forms of skin cancer.
Presently there is no cure for this disease. But there are promising approaches that could lead to successful treatments in the future. One of them is a method called “fibroblast injection”. In this procedure, fibroblasts are injected between the layers of the skin, where they can produce the necessary collagen 7.
Researchers at IMBA under the leadership of Arabella Meixner have now been successful in developing this method to treat mice affected by EB. The individual steps of this treatment have been worked out and carefully tested in many years of laboratory work, and the results have now been published in the scientific journal “Science Translational Medicine”.
First the scientists returned skin cells of the diseased mice to the stem cell stage and then repaired the genetic defect, the root cause of the disease. Then the researchers transformed stem cells back into fibroblasts.
Before the repaired fibroblasts could be reintroduced into the organism, measures to prevent inflammation or rejection were necessary. In this study the researchers conducted a type of “toxicity test”, and the results were very promising. After several months of observation, no adverse immune reactions occurred, and the risk of skin cancer did not increase. That is an important consideration because “butterfly children” already have a greatly increased risk of skin cancer.
But the greatest success of the researchers was the significantly increased tear resistance of the skin. Arabella Meixner, research lead, is delighted at the good results: “Our mechanical stress test with a soft eraser brush demonstrated that the skin of the mice treated with the stem cell therapy remained stable, and that no more wounds occurred. That means there was enough collagen 7 between the skin cells to hold them together properly. Our study clearly showed that this method is suitable for a future therapy for “butterfly children.”
The next step is to establish this skin stem cell treatment in humans. To achieve that, the IMBA scientists intend to look for partners with clinical experience. For severe forms of Epidermolysis Bullosa, a systemic application needs to be developed to spread the cells throughout the entire body via the bloodstream to reach epithelial tissues that are more difficult to access, for example the mucous membranes in the mouth or bowels. Often in “butterfly children” with milder forms of the disease, only certain areas of the skin are affected. The skin stem cell therapy with local injections successfully tested on mice could lead to a valuable treatment method in the very near future.
The project conducted by IMBA scientists was initiated by the patient organization DEBRA Austria, and has had the financial support of the association and of other generous supporters since 2009. DEBRA's mission is to ensure that “butterfly children” receive competent specialized medical care and to promote research into options to relieve and cure EB. Further thanks also go to our funding and cooperation partners „Österreichische Lotterien“ and „FK Austria Wien“.
Wenzel et. al., iPSC-based cell therapy for Recessive Dystrophic Epidermolysis Bullosa. Science Translational Medicine. 2014.
Dr. Arabella Meixner, Research Lead
Tel. +43 664 2018084
Evelyn Devuyst | idw - Informationsdienst Wissenschaft
North and South Cooperation to Combat Tuberculosis
22.03.2018 | Universität Zürich
Researchers Discover New Anti-Cancer Protein
22.03.2018 | Universität Basel
An international team of researchers has discovered a new anti-cancer protein. The protein, called LHPP, prevents the uncontrolled proliferation of cancer cells in the liver. The researchers led by Prof. Michael N. Hall from the Biozentrum, University of Basel, report in “Nature” that LHPP can also serve as a biomarker for the diagnosis and prognosis of liver cancer.
The incidence of liver cancer, also known as hepatocellular carcinoma, is steadily increasing. In the last twenty years, the number of cases has almost doubled...
In just a few weeks from now, the Chinese space station Tiangong-1 will re-enter the Earth's atmosphere where it will to a large extent burn up. It is possible that some debris will reach the Earth's surface. Tiangong-1 is orbiting the Earth uncontrolled at a speed of approx. 29,000 km/h.Currently the prognosis relating to the time of impact currently lies within a window of several days. The scientists at Fraunhofer FHR have already been monitoring Tiangong-1 for a number of weeks with their TIRA system, one of the most powerful space observation radars in the world, with a view to supporting the German Space Situational Awareness Center and the ESA with their re-entry forecasts.
Following the loss of radio contact with Tiangong-1 in 2016 and due to the low orbital height, it is now inevitable that the Chinese space station will...
Fraunhofer Institute for Organic Electronics, Electron Beam and Plasma Technology FEP, provider of research and development services for OLED lighting solutions, announces the founding of the “OLED Licht Forum” and presents latest OLED design and lighting solutions during light+building, from March 18th – 23rd, 2018 in Frankfurt a.M./Germany, at booth no. F91 in Hall 4.0.
They are united in their passion for OLED (organic light emitting diodes) lighting with all of its unique facets and application possibilities. Thus experts in...
A new scenario seeking to explain how Mars' putative oceans came and went over the last 4 billion years implies that the oceans formed several hundred million...
For the first time, an interdisciplinary team from the University of Basel has succeeded in integrating artificial organelles into the cells of live zebrafish embryos. This innovative approach using artificial organelles as cellular implants offers new potential in treating a range of diseases, as the authors report in an article published in Nature Communications.
In the cells of higher organisms, organelles such as the nucleus or mitochondria perform a range of complex functions necessary for life. In the networks of...
19.03.2018 | Event News
16.03.2018 | Event News
13.03.2018 | Event News
22.03.2018 | Trade Fair News
22.03.2018 | Earth Sciences
22.03.2018 | Earth Sciences