{"id":6020,"date":"2024-05-14T15:14:17","date_gmt":"2024-05-14T13:14:17","guid":{"rendered":"https:\/\/alpha1-deutschland.org\/?p=6020"},"modified":"2024-07-10T14:33:21","modified_gmt":"2024-07-10T12:33:21","slug":"3-alpha-1-patiententag-an-der-thorax-klinik-heidelberg-am-09-03-24","status":"publish","type":"post","link":"https:\/\/alpha1-deutschland.org\/en\/3-alpha-1-patiententag-an-der-thorax-klinik-heidelberg-am-09-03-24","title":{"rendered":"3rd Alpha-1 Patient Day at the Thorax Clinic Heidelberg on March 9, 2024"},"content":{"rendered":"<p style=\"font-weight: 400;\">Report from the 3rd Alpha-1 Patient Day at the Thorax Clinic Heidelberg<\/p>\n<p style=\"font-weight: 400;\">Dr.-Ing. Heinz Stutzenberger<\/p>\n<ol start=\"2\">\n<li style=\"font-weight: 400;\">Chairman of Alpha1-Germany eV.<\/li>\n<\/ol>\n<p style=\"font-weight: 400;\">\n<p style=\"font-weight: 400;\">On March 9, 2024, the 3rd Patient Day took place at the Thorax Clinic in Heidelberg, organized and conducted by Prof. Dr. Franziska Trudzinski. She is a senior physician in the Department of Pneumology and Respiratory Medicine, the deputy head of the General Interdisciplinary Outpatient Clinic, and for some time now a great asset to the Alpha-1 community as a scientific advisor for lungs, imaging, and gender medicine at Alpha1-Deutschland e.V. Together with the head of the Rhein-Neckar-Odenwald self-help group, Carsten B\u00fcch, and our chairwoman, Marion Wilkens, she welcomed all participants and thanked the event&#039;s sponsors, namely CSL Behring and Grifols, and the participating staff of the Thorax Clinic.<\/p>\n<p style=\"font-weight: 400;\">The welcome address was followed seamlessly by a presentation from Prof. Dr. Dr. Markus Ries, a specialist in pediatrics and adolescent medicine and, among other things, head of a research group for the development of drugs for rare diseases at Heidelberg University Hospital. His presentation addressed the question of how drugs for rare diseases are developed. These drugs, too, must go through the usual three phases of drug approval (see also the article &quot;Phases of Drug Approval&quot; in Alpha1 Journal 1\/2022 or on the Alpha1 Germany website).<em>,<\/em> However, they are confronted with several additional obstacles: there are usually few participants, the disease progression is often poorly understood, and the clinical picture is highly heterogeneous; there are no representative animal models; questionnaires for assessing quality of life are often only partially transferable; and finding study physicians is difficult \u2013 largely problems that will be familiar to Alphas. In the field of rare diseases, this must be addressed with a very well-thought-out trial process, with collaborative partnerships among all involved parties, including patients, and with the assistance of qualified trial centers specializing in the testing of rare drugs. The effort required for clinical trials is enormous, and many attempts are unsuccessful: of 10,000 substances in basic research, approximately 250 make it to preclinical trials, about 5 are clinically tested, and only one of these becomes a marketable drug. Historically, there are documented cases where it took 30 years for a research approach to result in a usable drug. Despite all these problems, even in the field of rare diseases, only those medications are approved whose safety from undesirable side effects and efficacy in improving health have been proven. When the associated costs must be distributed among only a few (rare!) patients, it helps manufacturers that their drug is protected from cheaper generic versions for at least a few years; in some cases, drug development is also funded by national or European institutions.<\/p>\n<p style=\"font-weight: 400;\">Following Professor Ries&#039;s lecture, Marion Wilkens and Carsten B\u00fcch shared some information about our association, along with details of the free(!) benefits that membership offers. These include a weekly memory training course, which many in the audience couldn&#039;t really picture. Carsten B\u00fcch then seized the opportunity by spontaneously highlighting an aspect of the training and circulating a word-finding exercise among the participants. This quickly revealed that the training was by no means a deadly serious affair; on the contrary, the participants were clearly having a lot of fun.<\/p>\n<p style=\"font-weight: 400;\">Following this, under bright sunshine and early spring-like temperatures, there was a walk through the &quot;Respiratory Path&quot; of the Thorax Clinic. This is a circular path in the clinic&#039;s spacious park, where several stations are set up for patients to independently perform movement, coordination, and circulatory exercises. These were eagerly used by the participants on Patient Day. The fact that a foosball table was also part of the equipment sparked genuine enthusiasm. Two mixed teams were spontaneously formed, each consisting of one doctor and one non-medical member. After the machine was coaxed into dispensing the necessary balls\u2014not by inserting coins, but by gently tapping it with fists\u2014the game began. It ended 5-1 for the blue team, although the red team was handicapped by a lame left-back. Afterwards, the participants strolled back to the conference building for lunch, engrossed in technical discussions, exchange of experiences or even the exchange of private topics.<\/p>\n<p style=\"font-weight: 400;\">Afterwards, work resumed: Professor Trudzinski gave a presentation on the topic of new treatment options for alpha-1 antitrypsin deficiency \u2013 what&#039;s in the pipeline? Fazirsiran was mentioned as the drug furthest advanced in clinical trials. It largely prevents the production of misfolded alpha-1 antitrypsin (AAT) in the liver. This reduces AAT polymerization in the liver, prevents the deposition of large agglomerates, and can even break down AAT that has already accumulated, allowing the liver to regenerate almost completely in the best-case scenario. The drug acts on a copy of a partial strand of the genome, thereby preventing it from being used as a &quot;template&quot; in the production of alpha-1 antitrypsin.<\/p>\n<p style=\"font-weight: 400;\">Another promising approach is recombinant AAT, which is no longer derived from human plasma but produced biochemically. Since this AAT is more stable than human AAT and therefore broken down more slowly by the body, its intravenous administration would be required at significantly longer intervals, e.g., only every 3 weeks (in contrast to the current augmentation with weekly infusions).<\/p>\n<p style=\"font-weight: 400;\">As a measure of the positive effect of drug treatment of the lungs, lung density measurement using computed tomography (CT) is currently replacing the measurement of difficult-to-reproduce lung function values such as FEV1, at least in clinical trials.<\/p>\n<p style=\"font-weight: 400;\">Further therapeutic approaches could emerge from the analysis of large databases, such as that of EARCO (European Alpha-1 Research Collaboration), which now comprises 1,300 data sets from Alpha-1 patients and has been used to demonstrate the effectiveness of augmentation by comparing the survival time of subjects from countries with and without this therapy. Unfortunately, the German contribution to this database is currently small, but it could increase in the future, and Professor Trudzinski encouraged those present to have their data included in the database through their treating physicians (for which, however, they require special qualifications).<\/p>\n<p style=\"font-weight: 400;\">A gender-specific study of individuals with alpha levels from the EARCO registry demonstrated that affected women suffer less frequently from COPD and liver problems than men, possibly due to a healthier lifestyle. However, symptom burden and the risk of exacerbations are the same.<\/p>\n<p style=\"font-weight: 400;\">The search for biomarkers, i.e. substances circulating in the blood whose concentration could provide an accurate prognosis about the expected course of the disease, is also still in an early stage of research.<\/p>\n<p style=\"font-weight: 400;\">Crucial for success in the search for new therapeutic approaches is the interdisciplinary collaboration of the various participating disciplines, i.e., the clinical medical profession, basic and applied researchers, and last but not least, the patients.<\/p>\n<p style=\"font-weight: 400;\">The final presentation of the day was given by Marion Wilkens on the question of why it is important for Alpha-1 patients to participate in studies. Two types of studies are important for Alpha-1 patients: firstly, registry studies (such as EARCO, see above), in which information on diagnoses, treatments, disease progression, etc., is collected and then made available for anonymized analysis. This allows researchers to identify correlations based on a large number of cases, which ultimately benefits the patients. Calls for participation in smaller studies are frequently posted on our association&#039;s website; entries in the EARCO database are made by qualified physicians.<\/p>\n<p style=\"font-weight: 400;\">Participation in clinical trials is considerably more involved, typically in Phase 3 of clinical testing, where a new drug is tested for efficacy in a controlled trial compared to a placebo. The undeniable risks are offset by a wealth of benefits, such as in-depth information on new treatment options, comprehensive medical consultation including a wide range of measurements and lab results not collected outside of studies, and, of course, the chance to benefit from a novel drug. However, one should not be pressured into participating but rather gather comprehensive information and then decide (although it is always possible to withdraw from a study if one feels uncomfortable). It is also possible that, as a participant in a study, one will not receive the new drug at all, namely if one is assigned to the placebo group (which is only revealed at the very end of the study).<\/p>\n<p style=\"font-weight: 400;\">Ms. Wilkens summarized all her points in one short sentence: Without the participation of Alphas in studies, there will be no cure.<\/p>\n<p style=\"font-weight: 400;\">Thus, the official part of a very informative and also exceptionally entertaining patient day drew to a close, but not without Carsten B\u00fcch expressing his hope that the target of 50 participants would be exceeded at the next patient day in 2025, which is hardly in doubt if the participants from 2024 pass on their extremely positive experiences to their acquaintances from the association.<\/p>\n<p><span style=\"font-weight: 400;\">Finally, a group photo was taken in front of Rohrbach Castle, a former hunting lodge in the neoclassical style located on the hospital grounds, which also included the aforementioned park. However, it took quite some time, during which the participants remained engrossed in conversation with each other and with the attending doctors and speakers, before the event finally came to a close.\u00a0 \u00a0<\/span><\/p>\n<p><img loading=\"lazy\" decoding=\"async\" class=\"alignnone wp-image-6021 size-large\" src=\"https:\/\/alpha1-deutschland.org\/wp-content\/uploads\/Pattag-Heidelberg24-1024x589.jpg\" alt=\"\" width=\"1024\" height=\"589\" srcset=\"https:\/\/alpha1-deutschland.org\/wp-content\/uploads\/Pattag-Heidelberg24-1024x589.jpg 1024w, https:\/\/alpha1-deutschland.org\/wp-content\/uploads\/Pattag-Heidelberg24-300x173.jpg 300w, https:\/\/alpha1-deutschland.org\/wp-content\/uploads\/Pattag-Heidelberg24-768x442.jpg 768w, https:\/\/alpha1-deutschland.org\/wp-content\/uploads\/Pattag-Heidelberg24.jpg 1481w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/p>\n<p>&nbsp;<\/p>","protected":false},"excerpt":{"rendered":"<p>Bericht vom 3. Alpha-1 Patiententag an der Thorax-Klinik Heidelberg Dr.-Ing. Heinz Stutzenberger Vorsitzender Alpha1-Deutschland e.V. Am 09.03.2024 fand der 3. Patiententag an der Thorax-Klinik Heidelberg statt, organisiert und durchgef\u00fchrt von&#8230;<\/p>","protected":false},"author":6,"featured_media":5142,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1,74],"tags":[],"class_list":["post-6020","post","type-post","status-publish","format-standard","has-post-thumbnail","category-allgemein","category-veranstaltungsberichte"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.3 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>3. 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