BARCELONA, Spain – Delegates from 23 countries discussed priorities for action as the 4th International Alpha-1 Patient Congress concluded here Saturday, April 13. The Congress marked the 50th anniversary of the discovery of Alpha-1 Antitrypsin Deficiency by Swedish researchers Carl-Bertil Laurell and Sten Eriksson in 1963. Eriksson attended the Congress and was honored for his landmark scientific achievement.
The delegates considered awareness of Alpha-1 to be the most important need for the Alpha-1 communities in their countries. Access to care, especially augmentation therapy, was the second most important priority named. The need for an organized Alpha-1 community in many countries and the need for better communication between Alpha-1 communities worldwide were also major concerns of the Alphas at the Congress.
John Walsh, president & CEO of the Alpha-1 Foundation, suggested an international organization to help support communications between Alphas around the world. He proposed to set up a website for the purpose, and promised to share the latest research information on augmentation therapy as soon as it becomes available.
Walsh said the Foundation would create a resource kit and share it with Alpha-1 organizations around the world, to support them in their own campaigns for access to augmentation therapy. “But it must be Alphas themselves who deliver this information,” Walsh said. “We in the United States had a major challenge to receive access to augmentation therapy in the 1980s and early 1990s,” he pointed out. “It took a concerted and united effort.”
More than 250 people attended the Patient Congress. The Congress, which was streamed online worldwide, began Friday with a video, “Alpha-1: 50 Years of Discovery,” featuring Eriksson and other leading Alpha-1 researchers reviewing the past, present and future of Alpha-1, including the possibility of a cure in the foreseeable future.
The opening ceremony and welcoming remarks included a talk by Eriksson himself, who gave the major credit to Laurell for first discovering the missing alpha-1 protein in electrophoretic strips for testing blood serum.
Alpha-1 groups from around the world displayed banners and talked individually about their countries and organizations at the Alpha-1 Global Village that followed.
Friday’s features included:
A panel of experts reviewing progress in scientific research over the past 50 years; “the patient perspective across the continents,” with Karen North representing Europe, Steven Knowles, Australasia, and Gordon Cadwgan, the Americas; expert panels on registries for patients and the status of testing in Europe and the USA; and a talk on “Alpha-1 Therapeutics Into the Future” by Mark Brantly, MD, director of the Alpha-1 research program at the University of Florida.
An Alpha-1 international research conference was held in conjunction with the Congress, and patients met in joint session with researchers on Friday afternoon. Patients raised key questions for the researchers at a Q&A session facilitated by a “Videowall” of questions recorded earlier, as well as questions from the meeting room floor.
Walsh used the same Videowall technology during his keynote speech Friday evening, playing back the responses from more than 20 Alpha-1 leaders from around the world answering the question, “What is the most important need for the Alpha-1 community in your country?”
Saturday’s agenda included:
A talk on “Health Technology Assessment” and its affect on reimbursement for treatment for rare diseases; the status of augmentation therapy; the AlphaNet model for self-care management; Alpha-1 resource centers; nutrition and inflammation; and lunch with the experts, a chance to eat at the same table as an expert on Alpha-1 and discuss questions and concerns.
The day ended with facilitated sessions on “Getting Things Done” for Alpha-1 patient groups around the world.
An international working group will be formed shortly to act on the priorities named by the delegates. “We’re all going to commit to each other that we’ll move ahead on these priorities,” Walsh said. “We all need to share our resources with each other.”
The Congress presentations were primarily in English, with some Spanish-English translation offered. Some delegates said that following the information and discussions was difficult for them, since many of the Alphas attending were native speakers of Portuguese, German, French, Swedish, Norwegian and other languages, and that translations in a variety of languages would be helpful
at future Alpha-1 Congresses.
This year’s International Research Conference on Alpha-1 focused on Alpha-1 liver disease for the first time. The plan is to hold an international Alpha-1 research conference every two years.