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            <title>News: HHU - Heinrich-Heine-Universität Düsseldorf</title>
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            <pubDate>Mon, 13 Apr 2026 06:00:47 +0200</pubDate>
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                        <guid isPermaLink="false">news-51752</guid>
                        <pubDate>Thu, 02 Apr 2026 10:00:00 +0200</pubDate>
                        <title>Understanding metastasis mechanisms</title>
                        <link>https://proxytest.hhu-d.de/en/news-article/understanding-metastasis-mechanisms</link>
                        <description>Metastases, i.e. the secondary growths of cancer cells, are a key problem in many types of cancer. As descendants of the primary tumour, they can grow in other organs distant from the primary site and are often difficult to identify. A research team from the Institute of Genetics at Heinrich Heine University Düsseldorf (HHU) has now identified molecular mechanisms, which can enable cancer cells to colonise other tissue. The team led by Dr Tobias Reiff has now published its findings in the scientific journal Nature Communications.</description>
                        <content:encoded><![CDATA[<p>Tumours develop when cells undergo such mutation that important functions are lost. These cells can then often divide indefinitely as the cell death mechanism is disabled or they are capable of bypassing the immune system. In most cases, if a tumour is discovered in good time, it can be treated: by means of operations, radiotherapy or chemotherapy – often in combination. Depending on the organ and type of tumour, the chances of recovery are often good.&nbsp;</p>
<p>The research group led by Dr Reiff is concentrating on colorectal tumours, which are often only discovered at a late stage. Reiff explains: “By that time, the cancer may have already spread, making it more dangerous: Individual cancer cells can infiltrate and colonise other tissue, where they grow and then destroy other vital organs.” <a href="https://www.krebsinformationsdienst.de/aktuelles/detail/metastasen-die-toedlichen-toechter" target="_blank" rel="noreferrer">According to the German Cancer Research Centre (DKFZ), around 90% of cancer patients die as a result of metastases rather than the primary tumour</a> (German only).</p>
<p>Reiff and his team are examining how cells separate from the primary tumour, spread via the bloodstream or lymphatic system (referred to as “dissemination”) and then colonise other tissue (“homing”). “These processes are not well understood. Contrary to formerly accepted wisdom, we now know that tumours can already spread at an early stage. Initially, however, the body’s own defence mechanisms can still prevent the spread of these disseminated cells successfully in most cases,” says Reiff.</p>
<p>The researchers have now identified signalling molecules called Netrins and the corresponding receptor (“Frazzled/Deleted in Colorectal Cancer”, for short: DCC), which control when the individual cells separate from the tumour and migrate. The fruit fly <i>Drosophila melanogaster</i> served as the research subject. It is particularly well suited as a model organism, as the flies have short lifecycles and reproduce on a daily basis.</p>
<p>“If we can gain an understanding of the underlying metastasis mechanisms in <i>Drosophila</i>, we may also be able to identify starting points for human therapies,” says Dr Lisa Zipper, lead author of the study in Nature Communications. There are great similarities at genetic level, e.g. large sections of the tumour-driving signalling pathways – which determine the frequency of division, cell death and cell fate – are the same.&nbsp;</p>
<p>The researchers in Düsseldorf are examining the flies using advanced laser microscopy techniques. Using proteins with fluorescent markings – which can be made visible on a selective basis via laser impulses – they traced how stem cells migrate and which molecules may be involved in metastasis. The researchers have named the new experimental approach developed for this work “Hamelin Assay” after the legend of the Pied Piper of Hamelin. It has enabled them to determine in detail how the migrating cells are guided through the intestine by the Netrins – like the rats in the legend are lured by the music from the rat-catcher’s pipe.&nbsp;</p>
<p>“This receptor molecule is altered in around 65% of colorectal cancer patients, which is a good indicator that the Netrins play a role in the spread of cancer,” says Reiff. Further studies are now needed to show whether the DCC receptor can be a starting point for future therapies aimed at countering metastasis.&nbsp;</p>
<p>The research work was funded by the Wilhelm Sander Foundation and the German cancer research/support organisation, <i>Deutsche Krebshilfe</i>.</p>
<h5>Original publication</h5>
<p>Lisa Zipper, Pol Ramon-Cañellas, Filiz Akkas-Gazzoni &amp; Tobias Reiff; Frazzled/DCC directs spatial progenitor integration ensuring steady-state intestinal turnover; Nature Communications 17, 2491 (2026)</p>
<p>DOI: <a href="https://www.nature.com/articles/s41467-026-70704-9" target="_blank" rel="noreferrer">10.1038/s41467-026-70704-9</a></p>]]></content:encoded>
                        
                            
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                        <guid isPermaLink="false">news-51721</guid>
                        <pubDate>Mon, 30 Mar 2026 10:24:07 +0200</pubDate>
                        <title>HHU President Anja Steinbeck signs agreements during a DAAD delegation trip to Ghana </title>
                        <link>https://proxytest.hhu-d.de/en/news-article/hhu-rektorin-anja-steinbeck-unterzeichnet-vereinbarungen-bei-daad-delegationsreise-nach-ghana</link>
                        <description>Within the framework of a university management delegation trip organised by the German Academic Exchange Service (DAAD), President Professor Dr Anja Steinbeck travelled to Ghana together with around 20 university management representatives from across Germany at the end of March. During the trip, she signed cooperation agreements with two Ghanaian universities – a further milestone in the growing collaboration between Heinrich Heine University Düsseldorf (HHU) and institutions on the African continent.</description>
                        <content:encoded><![CDATA[<p class="text-justify">On 23 March,&nbsp;Professor Steinbeck&nbsp;visited the University of Ghana in Accra, with which HHU has maintained links in various academic fields for years. This collaboration has now been formalised and deepened: President Steinbeck and Pro Vice-Chancellor&nbsp;Professor Dr Gordon A. Awandare&nbsp;signed a Memorandum of Understanding (MOU), which lays the foundation for the first partnership between HHU and an African university. At the same time, this represents the first institutionalised collaboration between HHU and a university in the Global South.</p>
<p class="text-justify">Plans are already particularly well established in the area of English Studies – where&nbsp;Professor Dr Birgit Neumann&nbsp;bears responsibility for the collaboration on the HHU side – and in the CEPLAS Cluster of Excellence on Plant Sciences (Plant Biology). The area of Global History is also involved:&nbsp;Professor Dr Stefanie Michels&nbsp;has coordinated Erasmus projects with the University of Ghana for many years and has already established various specialist contacts there. Talks were also held with the Pan African Doctoral Academy (PADA) in Accra.</p>
<p class="text-justify">“With the signing of this partnership, we are sending a clear signal: HHU understands international collaboration not as a one-way street, but rather as a genuinely mutual exchange on an equal footing. Ghana is an important partner for us – both academically and as part of our global responsibility as a university,” says President Professor Anja Steinbeck.</p>
<p class="text-justify">The visit also included a reception at the residence of the German ambassador in Ghana,&nbsp;Frederik Landshöft, which the entire university management delegation attended.&nbsp;Landshöft, who was born in Krefeld, is a HHU alumnus: He completed his bachelor’s degree in Social Sciences in Düsseldorf between 2003 and 2006, and was then involved in a research project funded by the German Research Foundation (DFG) as a student worker from 2005 to 2007 before gaining his master’s degree in International Relations and Development Policy at the University of Duisburg-Essen.</p>
<p class="text-justify">On 25 March, President Steinbeck travelled on to Kumasi, where the Kwame Nkrumah University of Science and Technology (KNUST) is located. HHU and KNUST – more precisely, the Kumasi Centre for Collaborative Research in Tropical Medicine (KCCR) based there – have already worked closely together in the field of tropical medicine for 15 years. President Steinbeck and the Vice-Chancellor of KNUST,&nbsp;Professor Rita Akosua Dickson, have agreed to extend the existing MOU and thus laid a new foundation for the institutional partnership. “Our partnership with KNUST is a perfect example of how sustainable academic cooperation can look. I am delighted that we are now continuing this long-term collaboration together,” says Steinbeck.</p>
<p class="text-justify"><strong>Strategic commitment in Africa</strong></p>
<p class="text-justify">The trip is part of HHU’s strategic commitment to establishing partnerships with universities in the Global South. “Africa is not a distant topic for HHU, but rather scientific practice brought to life. The trip undertaken by our President underlines the importance of these relationships to us,” emphasises&nbsp;Professor Dr Heidrun Dorgeloh, Vice President for International Relations at HHU.</p>
<p class="text-justify">HHU is a member of the Ghana-NRW University Alliance and operates a liaison office in Accra, the capital of Ghana, in cooperation with six other universities from North Rhine-Westphalia. Regular exchange formats at HHU such as the Round Table Africa support the growing collaboration with academic partners in Africa, which also includes a number of other countries on the continent.</p>
<p class="text-justify">&nbsp;</p>
<p class="text-justify">&nbsp;</p>
<p class="text-justify">&nbsp;</p>]]></content:encoded>
                        
                            
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                        <guid isPermaLink="false">news-51668</guid>
                        <pubDate>Tue, 24 Mar 2026 15:17:16 +0100</pubDate>
                        <title>The influence of lymph node architecture on lymphoma</title>
                        <link>https://proxytest.hhu-d.de/en/news-article/der-einfluss-der-lymphknoten-architektur-auf-lymphdruesenkrebs</link>
                        <description>Researchers at Heinrich Heine University Düsseldorf (HHU) and University Hospital Düsseldorf (UKD) have succeeded in mapping the organisation of immune cells in human lymph nodes for the first time. The study was conducted in collaboration with the German Cancer Research Centre (DKFZ), the European Molecular Biology Laboratory (EMBL) in Heidelberg, the Berlin Institute of Health (BIH) at the Charité hospital Berlin and the University of Basel. They were able to show why lymph node architecture is progressively broken down in the case of malignant lymphomas. The researchers, led by Professor Dr Sascha Dietrich (UKD), have now published the results in the scientific journal Nature Cancer.</description>
                        <content:encoded><![CDATA[<p>Lymph nodes are key control centres in the immune system and play an important role in defending the body against infections and tumours. For these processes to function properly, immune cells (B cells and T cells) must be organised in a precise spatial pattern in the lymph node tissue, for example in so-called B cell follicles and T cell zones. They are controlled by stromal cells (non-haematopoietic structural cells): They release messenger substances called chemokines, creating signals to guide the immune cells to their designated positions in the lymph node. In the case of B cell lymphomas, the internal structure of the lymph node tissue can be disturbed in very different ways, depending on the exact type of lymphoma: While the fundamental tissue structure remains intact in the case of slow-growing lymphomas such as follicular lymphoma (FL), aggressive lymphomas such as diffuse large B cell lymphoma (DLBCL) cause the tissue structure to break down completely. Why these typical growth patterns develop has been largely unclear to date.</p>
<p>In the study “Reprogramming of stroma-derived chemokine networks drives the loss of tissue organization in nodal B cell lymphoma”, the researchers coordinated by Professor Dietrich (Director of the Department of Haematology, Oncology and Clinical Immunology, UKD) have now succeeded in systematically mapping these processes in the human lymph node for the first time. By means of single-cell analyses and spatial tissue mapping, they were able to trace which factors lead to the progressive breakdown of the lymph node architecture in the case of lymphoma.</p>
<p>The data show: Stromal cells are the “architects” of the lymph node. The researchers were able to prove that central chemokine guiding signals in specialised stromal cells undergo fundamental changes over the course of lymphomagenesis, causing the progressive breakdown of the spatial structure of the lymph node. These stromal cell changes are reflected in the growth patterns of lymphomas – while there is a shift in the proportions of B cell follicles and T cell zones in FL, the areas remain spatially demarcated to a large extent; by contrast, there is widespread loss of important regulatory signals and thus tissue structure in DLBCL.</p>
<p>The study identified an inflammatory feedback loop as the driving mechanism: As part of the immune response in the tumour microenvironment, T cells produce inflammatory signalling proteins called interferons, which cause stromal cells to reprogramme their chemokine production: instead of structure-defining signals, inflammatory chemokines dominate, which in turn attract further inflammatory cells. The loss of lymph node organisation in lymphoma is thus not a passive effect of tumour growth, but rather actively driven by inflammation processes in the tumour microenvironment.&nbsp;</p>
<p>This reprogramming of stromal cells results in poorer patient survival rates. The study was able to demonstrate in large cohorts that a loss of structure-defining chemokines correlates with an unfavourable prognosis.</p>
<p>However, the findings also offer potential for new therapy approaches. “The study results show us that stabilisation of the stromal cells or targeted modulation of the inflammatory signals could be a promising new therapy approach,” says Professor Dietrich. “The findings could also help us identify new biomarkers in the future to enable early identification of aggressive disease progression,” he concludes.</p>
<p>The study took place within the framework of a national and international collaboration. In addition to Professor Dietrich and the Department of Haematology, Oncology and Clinical Immunology at UKD, leading partners included Dr Felix Czernilofsky (University Hospital Heidelberg), Dr Anna Mathioudaki (EMBL), Dr Lea Jopp-Saile (Max Delbrück Center for Molecular Medicine, Berlin), Professor Dr Simon Haas (BIH, Max Delbrück Center for Molecular Medicine, Berlin, Queen Mary University of London), Dr Daniel Hübschmann (DKFZ) and Professor Dr Judith Zaugg (EMBL and the University of Basel).</p>
<p>In Düsseldorf, Christina Schniederjohann (MSc), Dr Nora Liebers, Dr Peter-Martin Bruch, PD Dr Marc Seifert (all from the Department of Haematology, Oncology and Clinical Immunology) and Professor Dr Jörg Distler (Department of Rheumatology) were also involved.</p>
<h6>Full publication</h6>
<p><i>Architectural principles of lymphoma-induced lymph node tissue remodeling</i></p>
<p>F. Czernilofzky, A. Mathoiudaki, L. Jopp-Saile, R. Lutz, D. Vonficht, X. Wang, C. Schiederjohann, H. Voehringer, T. Roider, M.-A. Baertsch, C. Rodemer, H. Löffler-Wirth, M. Grau, D. Fitzgerald, J. Mammen, J. Kosla, N. Liebers, P.-M. Bruch, D. Ordoñez-Rueda, A. Brobeil, G. Mechtersheimer, C. Pabst, C. Müller-Tidow, A. Trumpp, M. Seifert, F. Neumann, M. Heikenwälder, C. Benes, W. Huber, J. Distler, G. Lenz, H. Binder, R. Siebert, G. P. Nolan, M. Gerstung, J. B. Zaugg, D. Hübschmann, S. Haas, S. Dietrich.&nbsp;<i>Nature Cancer</i> 2026.</p>
<p><a href="https://www.nature.com/articles/s43018-026-01136-z" target="Vollpublikation" rel="noreferrer">Full publication</a></p>
<p>DOI: 10.1038/s43018-026-01136-z10.</p>
<p>&nbsp;</p>]]></content:encoded>
                        
                            
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                        <guid isPermaLink="false">news-51664</guid>
                        <pubDate>Tue, 24 Mar 2026 15:11:00 +0100</pubDate>
                        <title>First ever antiproton transport</title>
                        <link>https://proxytest.hhu-d.de/en/news-article/erstmals-antiprotonen-transportiert</link>
                        <description>Today at the European Organisation for Nuclear Research (CERN) in Geneva, antiprotons have been transported across the site in a specially designed trap for the first time ever. This world premiere is a great success for the BASE collaboration, which is headed by Professor Dr Stefan Ulmer from Heinrich Heine University Düsseldorf (HHU). It is the first step toward transporting antimatter to other European laboratories. One such facility is the new high-precision laboratory being established at HHU, where the intention is to measure and compare the properties of antiprotons and protons with an unprecedented level of accuracy. </description>
                        <content:encoded><![CDATA[<p>The antiprotons were produced at the “Antimatter Factory” (AMF) at CERN, the only facility worldwide capable of producing these special low-energy particles. The BASE (Baryon Antibaryon Symmetry Experiment) research team has now succeeded in accumulating a cloud of around 100 antiprotons in a transportable trap known as a Penning trap. This trap was then disconnected from the stationary experimental facility, loaded onto a truck, then driven around the CERN site and finally reconnected to the experimental facility.&nbsp;</p>
<p>Professor Stefan Ulmer, BASE spokesperson and holder of the Chair of Quantum Technologies and Fundamental Symmetries at HHU: “A significant amount of know-how is required to confine and store antiprotons for a longer period of time, as antimatter annihilates immediately on contact with matter. For this reason, the antiparticles must be stored in an ultra-high vacuum using electric and magnetic fields to ensure they do not come into contact with residual gas particles or the storage vessel.”</p>
<p>But why make all this effort? The BASE collaboration is aiming to measure the properties of antiprotons, such as their intrinsic magnetic moment, extremely precisely and compare these results with those obtained for protons. BASE has long held the record for storing antiprotons for longer than a year at its stationary facility at the AMF.&nbsp;</p>
<p>However, to achieve even higher levels of precision, the physicists must overcome a problem: The accelerators at the AMF at CERN – where BASE is located – generate magnetic field fluctuations, which limit the degree of precision that can be achieved. “If we want to get a deeper understanding of the fundamental properties of antiprotons, we need an environment with less interference, which in turn means that we need to move out – for example to our laboratory for high-precision measurements of antiproton properties being established at HHU. That’s why we began designing a transportable trap around ten years ago and have developed it within the framework of the collaboration under the lead of Christian Smorra,” says Ulmer. The world premiere at CERN is an important test for this “relocation”: It demonstrates that it is technically possible to transport antiprotons to other European laboratories.</p>
<p>“Our aim with BASE-STEP is to be able to trap antiprotons and deliver them to precision laboratories at a dedicated space at CERN, to HHU, Leibniz University Hannover and maybe other laboratories, where the extremely precise antiproton measurements can be realised,” explains Dr Christian Smorra, member of Ulmer’s research group in Düsseldorf and head of the STEP project (Symmetry Tests in Experiments with Portable antiprotons) funded by the European Research Council (ERC). “We validated the feasibility of the project with protons last year, but what we achieved today with antiprotons is a huge leap forward toward our objective.” &nbsp;</p>
<p>BASE-STEP traps the antiparticles using magnetic and electric fields. The apparatus weighs around 850 kg. It can be loaded onto a truck, fits through standard laboratory doors and can withstand the jolts and vibrations during transport by road. It includes a superconducting magnet, liquid helium cryogenic cooling, power reserves and a vacuum chamber. This makes it significantly more compact than any other existing system for examining antimatter.</p>
<p>“To date, we have stored antiprotons loss-free in BASE-STEP for two weeks and can transport the trap autonomously for four hours,” says Smorra. “However, to reach our laboratory at HHU, it would take us at least ten hours. This means we’ll have to keep the trap’s superconducting magnet at a temperature below 8.2 K (-265°C) for that long.” Instead of liquid helium, which can run out, a generator will be needed to power a cryocooler on the truck.&nbsp;</p>
<p>“Transporting antimatter is a pioneering and ambitious project, and I congratulate the BASE collaboration on this impressive milestone. We are at the beginning of an exciting scientific journey that will allow us to further deepen our understanding of antimatter,” says Dr Gautier Hamel de Monchenault, Director for Research and Computing at CERN.</p>
<p>Professor Dr Anja Steinbeck, President of HHU, congratulated the researchers on their great success: “The scientific and technical feat achieved by the team headed by Professor Ulmer and Dr Smorra helps integrate research at major facilities and universities further. And lays the foundation for answering fundamental scientific questions in Düsseldorf – an extremely exciting prospect.”</p>
<h5>Matter, antimatter and the AMF Antimatter Factory</h5>
<p>For every particle of matter, there is an antimatter particle. They are virtually identical, apart from the fact that the charges and magnetic properties are reversed. According to the laws of physics, the Big Bang should have generated equal amounts of matter and antimatter. However, the particles and antiparticles should have quickly annihilated with each other to leave behind an empty Universe. Yet, the Universe comprises matter, meaning that an imbalance must exist. This has baffled researchers for decades. Physicists surmise that hidden differences exist, which can explain why matter ultimately survived and antimatter disappeared.&nbsp;</p>
<p>The AMF at CERN is the only site worldwide where low-energy antiprotons can be produced, stored and studied. Two so-called decelerators, the “Antiproton Decelerator” (AD) and the “Extra Low ENergy Antiproton Ring” (ELENA), supply several experiments with antiprotons. The lower the energy of the antimatter, the easier it can be stored for study purposes.&nbsp;</p>
<h5>The BASE collaboration and BASE-STEP</h5>
<p>The BASE (Baryon Antibaryon Symmetry Experiment) collaboration established in 2012 and based at the AMF at CERN, involves research institutes in Germany, Japan, the United Kingdom and Switzerland including:</p><ul><li>National Metrology Institute of Germany (PTB), Braunschweig</li><li>GSI Helmholtz Centre for Heavy Ion Research, Darmstadt</li><li>Heinrich Heine University Düsseldorf</li><li>European Organisation for Nuclear Research (CERN), Geneva</li><li>Leibniz University Hannover</li><li>Max Planck Institute for Nuclear Physics, Heidelberg</li><li>Imperial College London</li><li>Johannes Gutenberg University Mainz</li><li>RIKEN, Japan</li><li>University of Tokyo</li><li>Swiss Federal Institute of Technology in Zurich</li></ul><p>The founder and spokesperson of the collaboration is Professor Stefan Ulmer, holder of the Chair of <a href="https://www.antimatter.hhu.de/en/chair-prof-ulmer/dr-smorra-quantum-technologies-and-fundamental-symmetries" target="_blank">Quantum Technologies and Fundamental Symmetries at HHU</a>. He is also Chief Scientist at RIKEN in Japan.</p>
<p>Within the framework of the BASE collaboration, the STEP project – in which the transportable antiproton trap was developed – is funded by the ERC. This project is headed by Dr Christian Smorra.&nbsp;</p>
<p>More information: <a href="https://base.web.cern.ch/" target="_blank" rel="noreferrer">BASE website</a></p>
<h5>Video footage&nbsp;</h5>
<p>Explanation of how BASE-STEP works by Dr Christian Smorra: <a href="https://videos.cern.ch/record/3023672" target="_blank" rel="noreferrer">watch the video</a>&nbsp;</p>
<p>Report on the successful antiproton transport on 24 March 2026: <a href="https://videos.cern.ch/record/3023681" target="_blank" rel="noreferrer">watch the video</a></p>]]></content:encoded>
                        
                            
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                        <guid isPermaLink="false">news-51569</guid>
                        <pubDate>Wed, 11 Mar 2026 15:25:15 +0100</pubDate>
                        <title>New therapy approach for Leigh Syndrome</title>
                        <link>https://proxytest.hhu-d.de/en/news-article/neuer-therapieansatz-bei-leigh-syndrom</link>
                        <description>An interdisciplinary research team at Heinrich Heine University Düsseldorf (HHU) and University Hospital Düsseldorf (UKD) has, in collaboration with researchers from the Charité – Universitätsmedizin Berlin and the Fraunhofer Institute ITMP in Hamburg, identified a promising drug for the treatment of Leigh Syndrome. In the study, which has now been published in Cell, the researchers were able to prove that the drug Sildenafil had a beneficial effect on the course of the disease. The study was headed by Professor Dr Alessandro Prigione (Department of General Paediatrics, Neonatology and Paediatric Cardiology, UKD) and Professor Dr Markus Schuelke (Department of Pediatric Neurology, Charité).</description>
                        <content:encoded><![CDATA[<p>Leigh Syndrome is an inherited, progressive disease that affects the brain. It is a so-called mitochondrial disease, i.e. it affects the energy metabolism. The syndrome, which usually manifests in childhood, causes damage in the brain, which can lead to severe symptoms such as neurodevelopmental delay, epileptic seizures, muscular weakness and respiratory failure. No approved medication is currently available. The life expectancy of children with the disease is limited and most die within a few years of diagnosis.</p>
<p>With one case in 36,000 live births, Leigh Syndrome is classified as a “rare disease”. According to the European definition, a rare disease affects less than five in 10,000 people. The low number of cases makes research for treatments more difficult, resulting in a lack of approved therapies for many rare diseases. Another challenge is that these low patient numbers make larger-scale studies difficult. Studying Leigh Syndrome is further complicated by the lack of cellular or animal models, which can faithfully recapitulate the disease course of affected individuals.</p>
<p>The international research consortium set out to develop alternative model systems that can advance Leigh Syndrome research. In addition to HHU and the UKD, Charité and the Fraunhofer Institute for Translational Medicine and Pharmacology ITMP are also involved, together with several other research groups in Germany, Austria, Finland, the Netherlands, Poland, Italy, Greece and the USA. Headed by Professor Prigione, the researchers used skin cells from patients as a basis for deriving induced pluripotent stem (iPS) cells, which have the ability to differentiate into various somatic cells – for example nerve cells – in the laboratory. So-called brain organoids can also be derived from iPS cells, which represent 3D models of the brain. Patient-derived iPS cells served as an important foundation for the research findings, which have now been published.</p>
<p>On the basis of the nerve cells generated from patient stem cells, the researchers conducted an extensive drug screening process. They compiled a library of more than 5,500 drugs or drug-like molecules, which have in part been approved for other conditions and for which extensive safety and efficacy data are already available. These drugs were then tested on the nerve cells in the laboratory. In the course of the screening process, Sildenafil was identified as a potential therapeutic candidate. In the cell model, the researchers were able to prove that the drug had a positive effect on metabolism and improved the function of the cells affected by the disease. “With more than 5,500 compounds tested, this was the largest screening process to tackle Leigh Syndrome ever conducted. We are very proud that we have succeeded in realising this process and been able to identify a potential therapeutic agent”, says Dr Ole Pless&nbsp;(ITMP). Sildenafil is currently approved for the treatment of erectile dysfunction in adults. It is also approved for the treatment of pulmonary hypertension in infants. Sildenafil thus offers a good safety profile and promising results in terms of effectiveness in the cell model.</p>
<p>During the course of the study, it was also possible to confirm these results in the brain organoid and in different animal models, which encouraged the researchers to try Sildenafil as an individual basis treatment in six patients. The first Leigh Syndrome patient was treated with Sildenafil at Charité. Following positive results, further patients in Düsseldorf, Munich and Bologna were also treated. Sildenafil had a positive effect on the disease course in all patients treated. The medication was well-tolerated. “Extensive safety data on the long-term use of Sildenafil in children are already available, so we can assume that this could be a safe drug candidate for Leigh Syndrome,” reports Professor Prigione. “In the patients treated to date, we were able to observe that they recovered quickly from critical medical situations, their neurological function improved and their muscular strength increased,” adds Professor Schuelke (Charité).</p>
<p>As a result of these findings, the European Medicines Agency (EMA) has granted Sildenafil an Orphan Drug Designation (ODD). The ongoing Horizon research consortium SIMPATHIC, in which Professor Prigione and Professor Schuelke are also involved, is currently planning a multinational placebo-controlled clinical trial to determine whether Sildenafil is effective and safe in this patient population, and thus whether the EMA can approve it for the treatment of Leigh Syndrome. The publication is the result of a multinational collaboration within the framework of the CureMILS consortium funded by the European Joint Programme on Rare Diseases (EJP RD) and coordinated by Professor Prigione.</p>
<h6>Full publication</h6>
<p><i>Pluripotent stem cell-based screening uncovers sildenafil as a mitochondrial disease therapy</i><br>A. Zink, D-F. Dai, A. Wittich, M-T. Henke, G. Pedrotti, S. Heiduschka, G. Santamaria, T. M. Pentimalli, C. Brueser, S. Notopoulou, A. R. Umar, A. Zhaivoron, L. Petersilie, C. Jerred, J. Bergmans, L. A. Neu, F. Schumacher, J. Keller-Findeisen, A. Rybak-Wolf, D. Stach, J. Reinshagen, U. Haferkamp, K. Krieg, A. Zaliani, L. Euro, A. Di Donfranceso, C. Santanatoglia, E. Cappellozza, M. Suarez Cubero, M. Pavez-Giani, O. Bakumenko, D. Meierhofer, A. Foley, S. Morales-Gonzalez, I. Tolle, D. Herebian, D. Bonesso, G. Cecchetto, S. Nagumo Wong, M. Moresco, A. Maresca, I. Decimo, F. De Sanctis, A. Adamo, M. J. W. Adjobo-Hermans, R. Duchi, M. Barandalla, M. Scaglia, A. Perota, C. Galli, B. Kleuser, L, Cyganek, C. Mühlhausen, L. Schlotawa, V. Tiranti, E. Mayatepek, I. Szabo, C. La Morgia, T. Klopstock, V. Carelli, F. Distelmaier, A. Rossi, N. Rajewsky, G. Ullah, S. Jakobs, C. R. Rose, S. Petrakis, F. Edenhofer, W. J. H. Koopman, P. Lisowski, A. Suomalainen, D. Brunetti, A. del Sol, E. Bottani, O. Pless, M. Schuelke, A. Prigione.&nbsp;<i>Cell</i> 2026.</p>
<p><a href="https://www.cell.com/cell/fulltext/S0092-8674(26)00173-X" target="Full publication" rel="noreferrer">Full publication.</a></p>]]></content:encoded>
                        
                            
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