Author
Marion Wilkens, Alpha1 Germany eV, as published in Alpha1 Journal 2/2024.
About the ERS Congress
The ERS (European Respiratory Society) Congress is an annual conference that brings together leading experts in the field of respiratory diseases to present and discuss the latest scientific and clinical advances in respiratory medicine.
According to the pre-arranged program, our visit began with the ELF (European Lung Foundation) Patient Organisation Networking Day 2024. The focus here was on the exchange of information and transfer of experience with representatives of different countries and disease patterns.
The European Lung Foundation (ELF) is committed to ensuring that patient organizations and patient representatives have the opportunity to participate in the ERS Congress, contribute their valuable expertise to the program, and thus actively participate. A large portion of our travel expenses was covered by the ELF.
The daily program at the congress included scientific poster presentations and attending lectures. A highlight was an area showcasing numerous posters on the topic of alpha-1 antitrypsin deficiency. Many of these posters and reports were only made possible by providing our data for research – either through EARCO or directly from clinics. Young researchers giving presentations gave us hope for a future filled with new insights.
Special symposia organized by CSL Behring and Grifols, which aim to raise awareness about Alpha-1 deficiency and sensitize physicians, as well as sessions like the one held by EARCO, rounded out the program. At the latter, we were able to present patient-related projects to physicians and researchers.
The results protocol is presented very clearly below:
A special moment: The presentation of the ALTA Award
During the congress, the ALTAAward was presented, a research prize from the company Grifols, which is specifically dedicated to Alpha-1 Antitrypsin Deficiency.
The ALTA (Alpha-1-Antitrypsin Laurell's Training Award) was established in 2004 by Bayer HealthCare Biological Products and later continued by Talecris Biotherapeutics. Grifols has continued this commitment to the Alpha-1 community since 2011, aiming to foster the realization of innovative ideas and visions in the field of Alpha-1 antitrypsin deficiency and to provide a platform for novel research approaches. To achieve this, Grifols funds two research projects annually with prize money of €50,000 each.
Motivated clinicians and scientists are invited to apply for the ALTA Prize and submit their research projects. The two most promising projects, which provide new insights into the pathophysiology, clinical course, or treatment of AATD and related diseases, will be selected by a panel of international experts in the field of alpha-1 antitrypsin deficiency. The award ceremony takes place annually at the European Respiratory Society (ERS) Congress.
Awards 2024:
DR. BENOIT ALLARD: NEW THERAPEUTIC STRATEGY WITH ALPHA-1-ANTITRYPSIN-ENRICHED NANOPARTICLES
Dr. Benoit Allard received his PhD in Cell Biology and Pathophysiology from the University of Bordeaux in 2013. He is currently an Associate Professor at the University of La Réunion.
About the project:
In his project, he will evaluate the combined protective effects of apolipoprotein A1 nanoparticles (A1NPs), developed as therapeutic carriers (vectors), and alpha-1-antitrypsin (AAT) for the treatment of pulmonary emphysema in AAT™-associated tumor (AAT™) patients. This will be tested in a preclinical emphysema model through the inhalation of AAT-enriched A1NPs. Dr. Allard's team has demonstrated that native high-density lipoproteins (HDLs) can be loaded with AAT and that the intravenous injection of these nanoparticles significantly reduced elastase-induced pulmonary emphysema in mice compared to HDLs or AAT alone.
These nanoparticles possess anti-inflammatory properties. The aim of the project is to demonstrate a novel treatment approach for pulmonary emphysema in AATD through the inhalation of apolipoprotein A1 nanoparticles enriched with AAT (A1NP-AAT).
Background:
Intravenous administration of AAT allows only 2-3 % of the administered AAT to reach the lungs. While this does not halt disease progression, it slows the decline in lung function and density. However, it shows no effect on exacerbation rates or overall health status. To improve patient care, aerosol AAT therapy has been investigated. Inhalation allows replacement therapy to be delivered directly to the target site, achieving higher AAT concentrations. Recently, the first randomized, placebo-controlled clinical trial investigating the effects of inhaled AAT was completed. The study showed that inhaled AAT did not affect the time to first exacerbation in AATD patients with frequent exacerbations over 50 weeks, but may have influenced the pattern of episodes. Furthermore, the safety and feasibility of inhaled AAT in a large patient population were confirmed.
DR. TESSA SCHNEEBERGER: MENTAL DISORDERS IN COPD PATIENTS WITH AATD – PREVALENCE AND IMPACT OF PSYCHOTHERAPEUTIC INTERVENTIONS ON QUALITY OF LIFE
Dr. Tessa Schneeberger is a scientist at Philipps University of Marburg / Schön Clinic Berchtesgadener Land, specializing in non-pharmacological treatment options for chronic respiratory diseases, with a focus on improving pulmonary rehabilitation. Under the supervision of Prof. Dr. Rembert Koczulla and Dr. Inga Jarosch, her current research addresses the specific needs and personalized treatment options for COPD patients with acute respiratory distress disease (AATD). The department collaborates closely with Prof. Dr. Nikola Stenzel, an expert in chronic lung diseases and related mental health disorders, who also practices as a psychological psychotherapist (CBT).
About the project:
Their project investigates the prevalence of anxiety and depression symptoms in people with alpha-1 antitrypsin deficiency (AATD) and their impact on quality of life. It assesses the feasibility and impact of online psychotherapy on the quality of life of AATD patients with clinically relevant symptoms of anxiety or depression. Compared to people with chronic obstructive pulmonary disease (COPD), people with AATD experience an earlier onset of the disease. This early onset can contribute to reduced quality of life (QoL) and poorer mental health. Concerns about disease progression, genetic risks, lifelong treatments, weekly infusions, and early retirement represent significant psychological burdens. Mental health disorders are frequently underdiagnosed in people with AATD, even though they significantly affect quality of life and daily activities. Anxiety can lead to avoidance behaviors that limit physical activity and exacerbate symptoms such as shortness of breath and muscle weakness. To date, the prevalence of anxiety and depression in AATD remains unclear. Furthermore, there are significant challenges in implementing psychotherapeutic interventions for this patient group, particularly regarding accessibility and expertise. Few professionals are experienced in psychopneumology. Therefore, there is a clear gap in the understanding and treatment of the mental health of this population.
Pneumo Factory: Respiratory therapy for Alpha-1 patients
Since 2022, Dr. Schneeberger and Dr. Jarosch have been offering a personalized training and respiratory therapy program for people with AATD through their company, Pneumo Factory. Incidentally, contact with patients and support groups has revealed a significant need for information and treatment regarding mental health issues such as anxiety and depression within this patient group.
