Heinz Stutzenberger, as appeared in Alpha1 Journal 1/2025.

Three events from the Alpha-1 Europe Alliance appear newsworthy since the last issue of the Alpha1 Journal:

Firstly, there was the Alliance's introductory event for its partners, physicians, and sponsors, announced in Journal 02/2024. It took place in a Brussels hotel on November 15, 2024, under the title "Ensuring Equal Access to Treatment in Europe: Developing an Action Plan by 2030." Participants included representatives of the members and sponsors, as well as physicians with a long history of experience in the field of alpha-1 antitrypsin deficiency, such as Dr. Marc Miravitlles from Spain, Dr. Ilaria Ferrarotti from Italy, Prof. David Parr from England, Prof. Henedina Antunes from Portugal, and Dr. Stephanie Everearts from Belgium. The opening address, on the topic of "Ensuring Patient Participation in Policy Decision-Making Processes," was given by Valentina Botarelli, Head of Public Relations at EURORDIS, the European umbrella organization of patient organizations for rare diseases. The topics of "Barriers to Access to Treatment Options" and "Unmet Needs of Alpha-1 Patients" were then discussed in structured sessions and subsequently explored in greater depth in group discussions. The documented results are now being compiled and published in an Alliance action plan for the years 2025–2030, with support from the agency that organized and moderated the event.

Secondly, our involvement in applying for a research project within the framework of ERDERA, the European Research Alliance for Rare Diseases, which comprises national funding bodies for basic medical research, is worth mentioning. When we defined our priorities within the alliance before our official founding, including the item "Support for research projects on alpha-1 antitrypsin deficiency" seemed quite ambitious for a newly established organization. We were all the more surprised, therefore, when the initiators of such a project approached us with a request for our participation. Funding for research projects within the ERDERA framework has, among other things, two essential prerequisites: it must be a research consortium with participants from several European countries, and a patient organization must be involved—in this case, our alliance. The project involves university institutes in Italy, Belgium, the Netherlands, and Switzerland, and it focuses on basic research for a therapy that could one day be suitable for curing the lung tissue of alpha-1 patients, something long considered impossible. The project proposal was submitted on time, but unfortunately it was not selected over the other proposals. Now everyone involved is eagerly awaiting the reasons for the rejection so they can submit an improved proposal in the next round.

The final project to be reported on here is the campaign on the information channels of the Alliance and its members for Alpha-1 Awareness Day, which takes place annually on April 25th. The 2024 campaign, featuring eight different posts on various aspects of Alpha-1 antitrypsin deficiency and presented in the Alpha1 Journal 01/2024, was visually appealing and rather factual, in keeping with the tradition of the national Alpha-1 associations, and in retrospect, quite successful in terms of its reach. For the 2025 campaign, we benefited from the fact that our Italian colleague on the Alliance board works for a very large, international advertising agency, which, under a pro bono contract, agreed to develop a campaign for us free of charge (with a budget certainly in the five-figure euro range, which neither the Alliance nor the national associations could have afforded). Preliminary considerations regarding how to raise awareness for a rare disease amidst today's information overload led to a "disruptive" approach—one that involves profound changes to traditional methods. The presumed origin of Alpha-1 antitrypsin deficiency in the Viking Age was explored and spectacularly translated into short video sequences and striking images using AI. The aim is to generate enough interest in viewers to click through to a page where they can find further information about Alpha-1 antitrypsin deficiency in their own language and ultimately be directed to their national Alpha-1 association. If you haven't seen the campaign yet, please check the relevant information channels on the Alliance website. I hope to be able to report on the success of this campaign in the next journal.

Marion Wilkens, as appeared in Alpha1 Journal 1-2024.

The annual general meeting and information day in Austria took place at the Sporthotel Wagrain from May 24th to 25th, 2024. It is a special year for our colleagues and friends in Austria, as it marks the 20th anniversary of Alpha1 Austria.

As usual, the hospitality of our sister organization was wonderful, and I don't want to write a typical visit report today, but rather talk about friendship. From the very beginning, the exchange with our colleagues from Austria has been important to us. After so many years together, it's far more than just sharing the latest developments in the Alpha-1 world or joining forces in policy. True friendship has grown from it. Everyone has their own style and their own ideas, and that's the good thing—everyone can learn from each other. Through Renate Shashoua from the Swiss sister organization, our community has become even stronger and the friendship even closer. For me, the term DA-CH (Germany (D), Austria (A), and Switzerland (CH) represents an umbrella organization for patients in German-speaking regions and an open exchange, which is so valuable, especially when dealing with a rare disease.

We spent so many nights chatting and debating, and Alpha-1 was always the ultimate focus, sometimes our men too, but always secondarily. But they also bonded. Many know this from their own lives: much good and new can arise from a friendship, and so we wish our Austrian colleagues the next 20 years full of success, community, and always a good touch for the benefit of the patients.

Best wishes to Austria on the 20th anniversary.

The video about the anniversary and information day of Alpha1 Austria can be found here:

Marion Wilkens, Alpha1 Germany eV, as published in Alpha1 Journal 1/2022.

After much advance announcement about how important EARCO is, we have finally reached a milestone: Germany has enrolled its first patients in the European registry.

The Thorax Clinic in Heidelberg, under the direction of senior physician Dr. Franziska Trudzinski, has taken over the coordination of registry entries in the EARCO registry with the support of Dr. Timm Greulich (Marburg) and has already entered the first patient data. Further centers in Germany will follow and have already applied for accreditation.

What does this mean for me as a patient? I will undergo even more thorough examinations, as a large amount of data is collected for the registry. Since data collection must also be carried out regularly, I will receive an annual appointment. These annual checkups allow my doctor and me to detect any deterioration in my health as early as possible. This gives me the opportunity to better manage the illness through increased knowledge.

Why is such a register generally so important?
We want to understand why the course of the disease varies so much. Why, for example, do some affected individuals never become ill despite severe deficiency? Which comorbidities influence the severity of the disease? What has a positive effect on its course? To find out about such things, we need valid (good) data, ideally covering a longer period – and that's what the European registry is for.

Join in and actively ask your doctor about EARCO!

Marion Wilkens, Alpha1 Germany eV, as published in Alpha1 Journal 1/2022.

After a long drive, I arrived just in time for the wine tasting (a selection of fine wines for the next few days). It started off relaxed, and it was as if the pandemic had never happened. On Friday, we met early for the tasting, and only then did we receive our wristbands and name badges; everything was well organized.

The board team led by Ella Geiblinger was unanimously re-elected at the AGM, and we extend our warmest congratulations on behalf of Alpha1 Germany. We were given the opportunity to briefly present our organization and our work during the pandemic. Our collaboration with Alpha1 Austria has been close and very friendly for many years.

The event was very well moderated by Thomas J. Nagy; he created smooth transitions between the topics through short stories and involved us where appropriate.

The information day lived up to its name, as it offered a wealth of interesting information. It was beneficial to see the topic of advance healthcare directives from a medical perspective and the topic of long-term care benefits from the perspective of a lawyer (rather than a caseworker). This allowed the Austrians to be given many helpful tips.

Dr. Reiger presented her rehabilitation clinic; she proactively approached the Alphas in Austria, specialized in our condition, and strives to achieve the best possible outcome for us Alphas. Her vision:

International Research Conference and Global Patient Congress 2021

Alpha-1 Global

We are pleased to announce that registration for the International Research Conference 2021 and the Global Patient Congress is now open! You can now register for one or both events.

The International Research Conference will take place on Friday, the October 8, 2021 instead, and the Global Patient Congress on Saturday, the October 9, 2021.

Both conferences will be held virtually, allowing guests from around the world to participate. A live translation feature will be available, enabling participants to translate the presentations into 20 languages!

We look forward to interesting and informative meetings with the worldwide Alpha-1 community.