Author
Heinz Stutzenberger, as appeared in Alpha1 Journal 2/2024.
As readers of Alpha-1 Journal 1/2024 may recall, the Alpha-1 Europe Alliance got off to a dynamic start in its first year after its founding on October 17, 2023. This allowed for the successful launch of the major campaign for Alpha-1 Awareness Day on April 25. This momentum was largely thanks to our then-external coordinator, Veronica Lopez Gousset. To our great regret, however, she left us mid-year to pursue a doctoral degree at the Harvard THChan School of Public Health in Boston. Perhaps the Alpha-1 Europe Alliance board, by issuing a well-deserved and outstanding letter of recommendation, played a small part in enabling this significant career move.
The departure dealt a severe blow to the dynamics of the association, as the entirely volunteer board members, some of whom also work full-time jobs, had to search for a successor. Fortunately, one was soon found: Jill Bonjean has been the new coordinator of the Alpha-1 Europe Alliance since September 15, 2024. Originally from the USA, she now lives in Paris and has many years of experience with organizations working in the field of rare diseases. After securing funding for the Alliance, at least for the foreseeable future, by mid-year, the position of Communication Manager was also filled. Lila Martinez Ucha, who has Argentinian roots, now lives in Spain, and also has many years of experience in her field, was appointed to this role.
Of course, the association's activities did not come to a complete standstill even during the period without support. For example, two other organizations successfully completed the application process and were accepted as members:
- the Alpha-1 Foundation Ireland, whose representative had already been active in the founding preparations of the Alliance; however, due to a change in personnel, the application was delayed by several months.
- the Longfonds Foundation from the Netherlands, which is open to all patients with lung diseases and from whose ranks the Alphas sought admission to the Alliance.
The Danish Alpha-1 club, which had been a member of the Alliance for some time, merged with the clubs from Norway and Sweden in the summer and now represents all three national Scandinavian clubs as Alfa-1 Norden/Nordic.
At the time of its first anniversary (which, incidentally, was celebrated only virtually on its information channels), the Alliance now has 13 members and represents Alphas in 14 countries (the numbers are uneven, as there are two members in Spain and one member (Alfa-1 North, so) represents three countries). With one exception (France), all known national associations are now members of the Alliance, which thus represents approximately 4,400 patients.
This broad representation of Alphas across Europe is likely the reason why the Alliance is inundated with requests from pharmaceutical companies to establish various forms of collaboration with novel therapeutic approaches. These approaches are mostly still in a very early stage of development, often still undergoing preclinical trials. They range from novel chemical substances and interventions in RNA (in simplified terms, the template for the body's own production of AAT) to genetic engineering (in this context, it's worth noting that the first Alpha-1 patient in England was treated with such a drug in the middle of this year). Even if most approaches fail on the path to becoming a prescription drug, their sheer number gives hope that one or more developments will reach maturity, and it appears that the Alpha representatives at the European level will be able to support this development. Unfortunately, these discussions are subject to strict confidentiality agreements, so details cannot be published here.
It's not just pharmaceutical companies that are increasingly contacting the Alliance; we're also receiving a growing number of inquiries from patients who contact us via our online contact address. When these inquiries originate from member countries, we refer the patients to the national associations and facilitate direct contact. We handle inquiries from countries outside our member area ourselves, sometimes using translation software for questions and answers. Incidentally, the inquiry from the patient furthest away came from Chile! There has also been progress on the political front: at the sidelines of the SoHO conference of the European Health Union, where our member Bernd Dobbert advocated for more generous compensation for plasma donations (see his report in Journal 1/2024), the Alliance's chairwoman was asked by a representative of the European Commission to register the Alliance as a participant in further consultations on the regulation of "substances of human origin." Our application was approved, and since September 2024 we have been included in the list of organizations eligible to participate in meetings of the SoHO expert group. Therefore, should there be any changes or additions to the SoHO regulations in the future, we can ensure that our interests are heard in a forum where patient representatives are otherwise in the minority.
From September onwards, Jill and Lila began preparations for the two projects still planned for 2024.
On November 5, 2024, a large online patient conference was held, organized by Marion Wilkens and Karen O'Hara, secretary of the Alliance and chair of the Alpha-1 Association in the United Kingdom, together with the European Lung Foundation (ELF), the European Respiratory Society (ERS), and the Alpha-1 Registry EARCO. The event, spread over six hours, featured presentations on many relevant aspects of Alpha-1 antitrypsin deficiency, similar to the expert presentations at the Alpha-1 Germany information days, and also included a physical activity.
The lectures and presentations were streamed online in English, with subtitles available in 50 different languages. Both patients and doctors from many European countries participated. The scope of the lectures was far too extensive for an evening event, so it was held during the day, which unfortunately made participation difficult for working people. Nevertheless, more than 340 people from 35 countries registered. These came predominantly from Europe, with the furthest-traveled participants coming from Australia, and perhaps the most exotic country represented by a single participant was Eswatini, formerly Swaziland in southern Africa. The response to this event was overwhelmingly positive!
Considerable effort was also invested in preparing the Alliance's launch event with its partners, medical representatives, and sponsors. It took place on November 15, 2024, in a Brussels hotel under the title "Ensuring equal access to treatment methods in Europe: Developing an action plan by 2030".
Building on presentations by representatives of relevant organizations and the medical profession, parallel workshops with participants from all member organizations were held to outline ways in which the Alliance can contribute to achieving one of its main goals. I will report on the proceedings of this event and the results achieved in the next issue of the journal.
