Highlights of the 3rd International Research Conference on Alpha-1 Antitrypsin and the 6th Alpha-1 Global Patient Congress
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Highlights of the 3rd International Research Conference on Alpha-1 Antitrypsin and
the 6th Alpha-1 Global Patient Congress
The 3rd International Research Conference on Alpha-1 Antitrypsin and the 6th Alpha-1 Global Patient Congress took place from April 5-8, 2017, in Lisbon, Portugal. Both events brought together patients and scientists from around the world to discuss the latest developments in Alpha-1 Antitrypsin Deficiency (Alpha-1) research, clinical care and patient advocacy.
RESEARCH CONFERENCE (APRIL 5-6, 2017)
On April 5-6, 32 leading researchers and physicians met during the 3rd International Research Conference on Alpha-1 Antitrypsin. The meeting addressed novel treatment paradigms for Alpha-1 and other conditions that have become targets for Alpha-1 augmentation therapy.
During the first day of the conference, attendees had the chance to hear observations on the high throughput? of the liver cell model; the induced progenitor stem cells to study potential therapies; the roll of macrophages for the gene delivery; and the investigating liver therapies in the Alpha-1 Z Mouse. Presentations from the second day of the meeting focused on new treatments for lung and liver disease (such as intramuscular
and intra-pleural gene therapy; aerosol and antioxidant therapies; silencing RNA for liver disease and STC-GalNAc-conjugate approach to treat liver disease) and new disease targets for Alpha-1 Antitrypsin, including diabetes, organ rejection, autoimmune disorders and viral infections.
TRIBUTE TO JOHN W. WALSH (April 6, 2017)
After the closing of the Research Conference, patients, scientists, clinicians, industry partners and colleagues came together to express gratitude to Alpha-1 Foundation co-founder, John W. Walsh, who passed away last March. During the tribute, Walsh was recognized as an inspirational leader who dedicated his life to finding a cure for Alpha-1.
His personal dedication lead him to co-found the Alpha-1 Foundation and set the stage for Alpha-1 Global, a program to develop a collaborative global network of Alpha-1 patient leaders, physicians, and researchers, to increase awareness, detection, and access to care for Alphas around the world.
Walsh’s colleagues and friends from the international Alpha-1 community shared their personal experiences with him, strengthening their resolve to honor his legacy by continuing the work he started.
PATIENT CONGRESS (APRIL 6-8, 2017)
Following the Research Conference, the 6th Alpha-1 Global Patient Congress brought together Alpha-
1 patients and physician leaders from 34 countries. Patients and caregivers could interact with scientists and doctors who are leading clinical care and research to find better treatments for Alpha-1 patients, and ultimately to find a cure for Alpha-1. They also had the opportunity to engage with one another concerning the current needs of patients, and to update each other on activities including advocacy, education and self-management in the digital health era.
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Friday Program (April 7, 2017)
Morning session: research updates and global collaboration
Karen Skålvoll, a lung-affected Alpha and part of the Team Alpha-1 Athlete (Norway/Germany), officiated the Congress as emcee. During the two days of the Congress, she enlivened the sessions and encouraged the attendees to practice physical exercise as a necessary complement for the improvement of their health.
Following the official launch of the Congress by Gonny Gutierrez, Alpha-1 Global Director, the morning session was opened by Dr. Adam Wanner, Scientific Director of the Alpha-1 Foundation (US), who shared the latest research and therapeutic updates from the International Research Conference.
Professor Alan Altraja (University of Tartu, Estonia) spoke about the effectiveness of intravenous augmentation therapy, based on the positive results of the Rapid Trial and the Rapid-OLE Trial.
Sabina Janciauskienne (Hannover Medical School, Germany) presented “Alpha-1 Antitrypsin – Beyond Emphysema”, which focused on the possible development of chronic diseases that may follow the impaired biological activity of Alpha-1 Antitrypsin as a result of inherited or acquired deficiencies, as well as the need to learn better when and how to use Alpha-1 antitrypsin as a drug.
Professor Mark Brantly (University of Florida, Miami, US) highlighted the positive outcomes of the inhaled therapy for Alpha-1, such as its anti-inflammatory effect in lungs and the capacity of the aerosolized AAT to cross the interstitial space and reduce inflammation.
Marc Miravitlles (University Hospital Vall d’Hebron, Barcelona, Spain) and Pavel Strnad (University Hospital Aachen, Germany) provided updates on testing for Alpha-1 patients. They emphasized the importance of having reference centers and national registries for Alphas. They also stressed the need to motivate primary-care centers to use the resources at their disposal to test more patients who have Alpha-1 symptoms, in order to increase the likelihood of diagnosing more Alphas.
Marion Bouchecareilh (Bordeaux Research in Translational Oncology, France) focused her talk on the genetic mediators of Alpha-1 and the modifier genes of liver damage and emphasized, based on the validation in experimental systems of 2 candidate genes, the findings that provide promise for an early prognosis for AATD-associated liver disease which could even lead to possible pharmacologic developments.
The morning session finished with a panel on Research and Global Collaboration, which was facilitated by Gonny Gutierrez, Alpha-1 Global Director. She summarized the outcomes of the Global Alpha-1 Investigator and Physician Forum, held in Dublin on November 2-3, 2016. Organized with the purpose of exploring the feasibility, structure and function of a global network of Alpha-1 researchers and clinicians, the forum attendees created an inventory of possible collaborative efforts to streamline Alpha-1 research & physician efforts around the world within the following categories:
- Physician Education to allow the establishment of global criteria for centers of excellence, including criteria for expert certification and globally accepted reference labs in Alpha-1 Antitrypsin Deficiency (AATD); global guidelines for diagnosis and treatment (reconciliation of existing guidelines) and possibly a global online consultation service for patients.
- Patient Registries Collaboration to promote the development of national registry standards and a communication tool for patients who are not currently in a registry. The project would allow the collection of patient-reported questionnaire data. It would also quantify and define exacerbation in AATD.
- Alpha-1 Research Collaboration to establish global lab-testing standards; create globally positioned centers of excellence; advocate for funding sources in Europe; and conduct a prevalence study and detection program in countries lacking these resources.
- Funding for Research to raise funds for continued research into Alpha-1 and novel or emerging therapies at a global level. The Forum discussed how the developing network worldwide could help achieve a more sustainable future.
As starting project resulting from the Forum, Robert Sandhaus (Alpha-1 Foundation Medical Director, Jewish Health, Denver, CO) discussed how the development of a global Guidelines Initiative to reconcile existing and developing guidelines on the treatment of Alpha-1 is underway.
The Alpha-1 Foundation is already working on an upgraded Research Registry format that will include patient-reported data collection worldwide. The Foundation is also looking at the next steps, such as working with Australia on harmonizing or integrating their collected Alpha-1 data, so that each country can share its data more easily with others.
Afternoon session: access to therapies, advocacy and organ transplantation
The afternoon session was opened by Sandra Nestler-Parr (Roboleo and Co, Alpha-1 UK Support Group), who outlined the steps needed to overcome barriers to augmentation therapy access. She also challenged patient organizations, clinicians and industry partners to work more closely to ensure better use of collected patient data. Health technology assessors require this data to provide recommendations on medicines and other health technologies that can be financed or reimbursed by the various European healthcare system.
Charles Waller (Rohde Public Policy Health Brussels, Belgium) discussed the development of the EU Policy Recommendations for Alpha-1 Antitrypsin and the current public policy approach at the European Parliament level within the rare disease space. Robert Stockley (Queen Elizabeth Hospital, Birmingham, UK) led a session on concerted efforts within clinical practice advancement.
Sleep related issues were addressed by Dr. Leonardo Fuks (Rabin Medical Center, Petah Tikva, Israel) who pointed out the effects of insomnia and sleep apnea.
Rosa Malo de Molina Ruiz (University Hospital Puerta de Hierro Majadahonda, Spain), a pulmonary transplant specialist, discussed the current procedure for pre and post lung transplant patients. She emphasized the key concerns and needs of patients and healthcare professionals involved in transplants.
A panel discussion on lung and liver transplantation closed the afternoon session led by Karen
Erickson (Alpha-1 Foundation, Miami, US). The panel brought together both lung and liver transplanted patients (including children) who shared their personal experiences and concerns. They highlighted the importance of being psychologically well-prepared before the transplantation procedure. They also emphasized the need for strong support from relatives, caregivers, friends and a transplant team in which they feel confident.
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Saturday Program (April 8, 2017): exercise, e-health, patient advocacy and global commitment
The second day of the Patient Congress started with a dynamic session led by Karen Skålvoll, who encouraged the attendees to maintain an active daily life with simple fitness tools and exercise plans. She also challenged other Alphas to improve their physical activity levels as “Alpha Warriors” and explained how she, a seasoned athlete, marathon runner, and oxygen user, has overcome barriers to continue participating in leading sporting events worldwide, and to help raise awareness and funding for Alpha-1 research and related programs.
Shane Fitch (Lovexair Foundation, Spain and Portugal) provided a summary of what digital healthcare apps and web-based disease management care programs mean to the future of the patient community. She pointed out that such technology could improve patients’ self-care and produce better health outcomes for each patient.
Moving forward on advocacy for those countries that still have no access to care, Karen O’Hara (Alpha-1 UK Support Group,Worcestershire) gave a concise simple step plan and pointed out the key considerations that patient leaders need to work on to progress effectively. This roadmap has been used in the UK with considerable input from experts in public policy.
Frank Willersinn (Alpha-1 Plus Brussels, Belgium) talked to new country members about how to network with specific global, European and other organizations to strengthen their positioning and capabilities to achieve their objectives. Willersinn noted that once the organizations have established what their objectives are, the Alpha-1 Global network could help by connecting people and providing training and support.
Joanna Chorostowka (National Institute of Tuberculosis and Lung Diseases, Warsaw, Poland), 
Ruxandra Ulmeanu (Institute of Pneumology, Bucharest, Romania) and Aleksandra Ilić, (Medical School University Belgrade, Serbia) closed the morning sessions, sharing their experiences as Central & Eastern European physicians who spearhead patient collaborations on a national level in their respective countries.
Congress closing: Global commitment and next steps
An interactive panel session with Steering Committee Members closed the morning session by addressing questions and comments brought up by the audience.
Afterwards, Gonny Gutierrez presented Alpha-1 Global’s strategic direction for 2017-2010, which has as parameters the 3 pillars derived from the program’s mission statement:
Goal 1 – Collaboration: facilitate ways to effectively collaborate between patient leaders, physicians, researchers and any other stakeholders on a global level.
- Biennial Alpha-1 Global Patient Congress (2017 and 2019)
- Regional Training and Collaboration
- Europe, LATAM and Australia/New Zealand
- EU Policy Recommendations
- Scientific research collaboration
- Global guidelines document development
- Integration of existing research registries
Goal 2 – Materials, Training and Communiqués: provide the global community with Alpha-1 related educational materials, updates and strategic training for use at a local level.
- Lisbon follow-up questionnaire:
- Keep global information current
- Identify where expert training is needed (globally/online or specific to a country)
- Create manual for new Alpha-1 Support Groups
- Continued website, bi-monthly eNews publications and social media presence
Goal 3 – Network Expansion: expand the global network of Alpha-1 stakeholders to build capacity in local communities and contribute expertise to the alpha-1 global community at large.
- Assist with starting new patient organizations
- Expand network of A1 patient community representatives and trainers
- Alphas who can serve in a wider capacity as “experts” in their field and be part of case studies
- Expand global network of physicians and researchers
- Actively grow our physician and researcher database and expand collaborative efforts worldwide
Henry Moehring (Alpha-1 Foundation, Miami, US) officially closed the Patient Congress with a commitment of the Alpha-1 Foundation to support Alpha-1 Global’s activities around the world.
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