Alpha-1-To-One Magazine | Vol. 17, No. 1 (Spring 2019)

Vol. 17, No. 1 • SPRING 2019

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A MAGAZINE OF THE ALPHA-1 FOUNDATION

ALPHA- -TO-ONE Practical advice, personal experiences and

pertinent news for people touched by ALPHA-1

Honoring a legacy: Moving the mission forward PAGE 4

The Alpha-1 Foundation’s response to the COPD National Action Plan PAGE 6 Near and far, each step brings us closer to a cure PAGE 17 Honoring Jim Quill PAGE 19

LETTER FROM THE PRESIDENT

Honoring

a legacy and moving the mission forward

A

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ALPHA- -TO-ONE Practical advice, personal experiences and pertinent news for people touched by Alpha-1, their families and friends VOL. 17, NO. 1 (Spring 2019) Published by the Alpha-1 Foundation 3300 Ponce de Leon Blvd., Coral Gables, FL 33134 (877) 2 CURE A1 (228-7321) www.alpha1.org Alpha-1 Foundation Board of Directors Executive Committee Jeanine D’Armiento, MD, PhD, Chair Elizabeth Johnson*, Vice Chair Jennifer Jopp*, Secretary Fred Walsh*, Treasurer Gordon E. Cadwgan, PhD*, Immediate Past Chair Members Virginia Clark, MD Kenneth Irvine + Peggy Iverson* Ann Knebel, PhD, RN Noel G. McElvaney, MD, MCh, BAO, FRCPI, FRCPC James K. Stoller, MD, MS (Org. Dev.) Frank Willersinn, MD* Martin R. Zamora, MD, Chair of MASAC Alpha-1 Foundation Executive Staff Miriam O’Day, President and CEO Mark B. Delvaux, Vice President and CFO Adam Wanner, MD, Scientific Director Robert A. Sandhaus, MD, PhD, FCCP, Clinical Director Alpha-1-to-ONE Editorial Board Jen Childress +; William J. Martin II, MD; Robert A. Sandhaus, MD, PhD, FCCP; Bruce C. Trapnell, MD Editor-in-Chief & Executive Editor Miriam O’Day Managing Editor Jeanne Kushner Editorial Manager Alejandro E. Hernandez Padilla Contributing Editors Alexis Artiles Ojeda; Barbee Bennington; Adriana De Arce; Cathey Horsak; Richard Lovrich; Angela McBride; Randel Plant; Maddie Requejado; Linda Rodriguez

s we take a look into what has been accomplished by the Alpha-1 Foundation in the past decades, it is imperative that we honor the legacy and continue Honoring a legacy and moving the ofmission the efforts that my friend, mentor and co-founder the Foundation, John W. Walsh, started.

forward

As we take a look into what has been accomplished by the Alpha-1 Foundation in the past

The work that have to do together carrythe forward andand continue the efforts that my friend, decades, it isweimperative that wewill honor legacy forever memorialize the innovative theWalsh, started. mentor and co-founder of thecontributions Foundation,made JohnbyW. Foundation under his leadership.

The that wemilestones have to do carry As wework see important andtogether advances will taking placeforward and forever memorialize the by the Foundation under his leadership. ininnovative research andcontributions detection, we atmade the Foundation must remain centered in scientific research, which will ultimately lead us milestones advances have taken place in research and detection, we at toAlthough fulfill the important core of our mission: to findand a cure for Alpha-1 the Foundation must center our efforts in scientific research, which will ultimately lead us to Antitrypsin Deficiency (Alpha-1). When that moment comes fulfilling the core of our mission: to find a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1), and and history is kind enough to lead us there, the job will be when that moment comes and history is kind enough to lead us there, the job will be far from far fromasover the important tasks detection, spreading spreading awareness and improving the lives of all over theasimportant tasks of of detection, awareness and improving lives ofcondition, all those affected by this will remain our focus. those affected by thisthe genetic worldwide, genetic condition, worldwide, will remain our focus.

You,the theAlpha-1 Alpha-1 community, themotivation main motivation of the Foundation and will remain You, community, havehave been been the main ALPHA-1-TO-ONE is published by the Alpha-1 Foundation with support the main focus of our work. of the Foundation and will remain the focus of our work. from its advertisers. No part may be reproduced in any form by any means without prior written permission of the Alpha-1 Foundation.

The contents are not intended to provide medical advice, which I am grateful to be selected to serve the Alpha-1 Foundation be obtained directly from a physician. The Foundation is I am grateful to be selected to serve the Alpha-1 Foundationshould you, for our as expressed in notand responsible the constituents, accuracy of information and you, our constituents, as president and CEO. Thank you to advertisements in this publication.

President and CEO. Thank you to all of you who have supported and guided me upon my return Advertising: For advertising inquiries, please contact Angela McBride, all youorganization. who have supported and guided meyou uponhave my return toofthe It is an honor that put your faith in ofme, andRelations I promise serveext.you Director Corporate at (877)to 228-7321 233, or amcbride@alpha1.org. towith the organization.

It is an honor that you have put your faith distinction.

Letters to the Editor: Please send letters to the editor to jkushner@alpha1.org, Director of Communications. Letters may in me, and I promise to serve you with distinction. be edited for clarity and length.

ALPHA-1 1 FOUNDATION Miriam O’Day

Miriam O’Day

President President&&CEO CEO

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The Alpha-1 Foundation is committed to finding a cure for Alpha-1 Antitrypsin Deficiency and to improving the lives of people affected by Alpha-1 worldwide. *Diagnosed with Alpha-1 Antitrypsin Deficiency + Diagnosed Family Member

1-877-2-CURE-A1 (228-7321)

­­­Inside

Vol. 17, No. 1 • SPRING 2019

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A MAGAZINE OF THE ALPHA-1 FOUNDATION

­­­ALPHA- -TO-ONE

Features

SPOTLIGHT Miriam O’Day Alpha-1 Foundation welcomes new president and CEO ......................................................................... page 4

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FRONTIERS The Alpha-1 Foundation’s response to the COPD National Action Plan 17th Gordon L. Snider Critical Issues Workshop provides recommendations .............................................. page 6 EARCO: European Alpha-1 Research Collaboration Creating a European Alpha-1 Antitrypsin Deficiency Registry and network............................................ page 8 Alpha-1 research studies enrolling The latest Alpha-1 studies enrolling volunteers through the Alpha-1 Research Registry ............................ page 28 ALPHA LIFE A generous gift matched, honoring two decades of excellence The Cadwgan family honors Robert C. Barrett, AlphaNet matches donation .......................................... page 10 Honoring Jim Quill Remembering Alpha-1 leader, Jim Quill ............................................................................................... page 19

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ALPHA-1 KIDS A mother’s perspective Joan Garry’s journey as an Alpha-1 parent......................................................................................... page 13 IN YOUR INTEREST “The path to finding a cure,” new scientific track at National Conference Alpha-1 experts join to share latest advancements towards a cure............................................................ page 15 Near and far, each step brings us closer to a cure The community comes together to spread awareness................................................................................. page 17

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ASK THE ALPHA DOC Robert A. Sandhaus, MD, PhD, and Charlie Strange, MD, answer questions from the community........... page 20 ALPHA-1 COMMUNITY Ric Logsdon: “I have Alpha-1 but Alpha-1 does not have me” AlphaNet coordinator shares his Alpha-1 story.................................................................................... page 23 Alphas in the heartland Support group leaders keep up the good work......................................................................................... page 24

CALENDAR 2019 Support group meetings, events and education days ................................................................................ page 27

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28th Annual Alpha-1 National Conference The 2019 conference is set to take place June 21-23 in Orlando, Florida ................................................ Center Spread Cover photo by Richard Lovrich

fb.com/alpha1foundation www.alpha1.org

@AlphaFriend

@alpha1foundation­­­

@Alpha-1FDN 3

SPOTLIGHT

Alpha-1 Foundation welcomes new

president and CEO T

he Alpha-1 Foundation board of directors announced the appointment of Miriam O’Day as the new president and chief executive officer (CEO) of the Alpha-1 Foundation on Feb. 1, 2019.

O’Day served as interim CEO beginning fall of 2018, working closely with all stakeholders to move the mission forward.

“We thank Miriam for serving as interim CEO and appreciate her dedication to the organization and hard work during the transition. The board of directors welcomes Miriam as a perfect fit for the Alpha-1 Foundation, as the new president and CEO. Her experience with the Alpha-1 patient community, the research and medical community, and public policy at the federal level extends nearly 20 years and will help lead our Foundation forward,” said Jeanine D’Armiento, MD, PhD, chair of the board of directors of the Foundation.

O’Day and the late John W. Walsh first met in the late 1990s, when she was vice president of the Immune Deficiency Foundation (IDF). She and Walsh worked together for years on blood safety issues that were critical to both of them, since the immune deficiency and Alpha-1 communities both depend on plasma-based therapies.

When O’Day left the IDF in 2000, Walsh, co-founder, president and CEO of the Alpha-1 Foundation at the time, contracted her to serve as the Foundation’s senior director of public policy.

“Miriam has done the impossible,” said Walsh while introducing her as keynote speaker at the 2015 Alpha-1 National Conference, in

Garden Grove, California. “She built a special, effective relationship called a liaison relationship with the U.S. Food and Drug Administration (FDA) and the National Heart, Lung, and Blood Institute (NHLBI). She engaged with the Centers for Medicare and Medicaid Services (CMS) on issues such as supplemental oxygen and access to pulmonary rehab. She’s carried the torch for us on regulatory affairs – the best patient advocate any community could possibly have.”

For years, O’Day had her own consulting business where she represented many organizations besides the Foundation, including the former Alpha-1 Association, the COPD Foundation, the U.S. COPD Coalition, nontuberculous mycobacteria (NTM) lung disease Info and Research, and the American Association for Respiratory Care (AARC).

O’Day pushed forward the idea of a Foundation magazine, which she described as “a People magazine for Alphas.” This idea became the now widely circulated, international publication called Alpha-1-To-One. She was the first editor, and in 2008, she was on the cover as the spotlight feature of the winter issue, which focused on her advocacy and public policy work.

She was the voice for people with lung diseases and the allied health professionals who care for them (respiratory therapists), on Capitol Hill.

O’Day looks forward to continuing the work and honoring the legacy left by Walsh, to move the mission forward; to find a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1) and improve the lives of those affected by the condition worldwide.

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1-877-2-CURE-A1 (228-7321)

Photo: Richard Lovrich

“John W. Walsh was an inspirational person who engaged everyone he met to join the Foundation’s mission. There are so many ways he was a trailblazer in rare disease research and therapeutic development,” expressed O’Day. “The Foundation’s success sits squarely on the shoulders of John and the people who joined with him, as staff, volunteers, scientists, ethicists, clinicians, patients and family members. I will work hard to continue John’s legacy and prioritize Alpha-1 among all stakeholders,” she FOUNDATION finished. 1 ALPHA-1

Miriam O’Day President and CEO of the Alpha-1 Foundation

Miriam O’Day has been involved in many key achievements for the Alpha-1 and rare disease communities, including: • “March on Washington” that persuaded Medicare to cancel planned reimbursement cuts for augmentation therapy.

• Helping to win passage of the Rare Disease Act that led to the establishment of both the Rare Lung Disease Consortium and the Cholestatic Liver Disease Consortium – both of which invest research dollars into Alpha-1.

www.alpha1.org

• The Genetic Information Nondiscrimination Act (GINA), which bans the use of genetic information in health insurance and employment decisions.

• The CMS decision that Medicare would cover pulmonary rehabilitation.

• The U.S. Department of Transportation ruling that required all airlines operating in the United States to allow passengers to take approved portable oxygen concentrators with them on the plane as carry-on.

• The Patient Notification System to notify patients of any recalls of blood products they might be using.

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FRONTIERS

The Alpha-1 Foundation’s Response

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n 2017, the National Institutes of Health (NIH) and the National Heart, Lung, and Blood Institute (NHLBI) published the “COPD National Action Plan.” The plan was the result of Congressional recommendations that the NIH, and NHLBI should address chronic obstructive pulmonary disease (COPD), the third leading cause of death in the U.S., with input from all stakeholders. A town hall meeting was held with patients, voluntary health organizations, clinical investigators, health care providers, industry, the Food and Drug Administration (FDA), the Centers for Disease Control and Prevention (CDC) and representatives from other institutes of the NIH. In response to the COPD National Action Plan, the Alpha-1 Foundation sponsored the 17th Gordon L. Snider Critical Issues Workshop, “The Alpha-1 Foundation’s Approach to the COPD National Action Plan.”

This one-day workshop identified areas in which the Foundation can effectively promote programs in the spirit of the COPD National Action Plan, but with a focus on the leading identified genetic risk factor for developing COPD, Alpha-1 Antitrypsin Deficiency (Alpha-1).

The workshop was held in New York City on Nov. 15, 2018, and included speakers and discussants from the NHLBI, COPD Foundation, academia, patient community, and the Alpha-1 Foundation.

James Kiley, PhD, director of the Lung Division of the NHLBI, opened the meeting by reviewing the origin and development of the COPD National Action Plan and emphasizing

the need for program implementation

by COPD-targeting voluntary health organizations, which include the Alpha-1 and COPD Foundations.

It was the intent of the COPD National Action Plan to address the needs in detection, treatment, and management of COPD, and to energize the COPD community to develop and implement specific programs to address these areas of need.

The topic of the first session was the role of exacerbations in COPD and included presentations by Byron Thomashow, MD, from Columbia University, and Michael Campos, MD, from the University of Miami.

Thomashow stressed the point that while we have learned a lot about the clinical significance of exacerbations as well as medical interventions to prevent them from occurring, the methodologies of managing exacerbations have not changed in decades. While Campos reported that the frequency, severity, and management of COPD exacerbations in Alpha-1 are similar to those reported for COPD in general, based on his observational data in a subset of over 1,000 of the Alpha-1 patients enrolled in the AlphaNet disease management program.

Therefore, more information is needed on the preventive and therapeutic effect of augmentation therapy on COPD exacerbations in patients with Alpha-1.

In the second session, entitled “COPD-targeted Detection of Alpha-1 Antitrypsin Deficiency,” a patient’s experience was contrasted with the healthcare provider’s view. Elliott Kassoff, an individual with Alpha-1-related COPD, shared his personal story that highlighted the years that elapsed between his onset of symptoms and his diagnosis of Alpha-1, and the number

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to the COPD National Action Plan of doctors involved to finally detect the condition.

Franck Rahaghi, MD, from the Cleveland Clinic, reviewed the currently available outcome data from diverse COPD-targeted detection studies, in view of several practice guidelines that strongly recommend that all patients with COPD should be tested for Alpha-1 regardless of ethnic background, gender, age or smoking history. Such studies include the former State of Florida sponsored program promoting medical office-based detection, electronic medical record-based testing reminder approaches, pulmonary function laboratory-based testing and the Alpha-1 Foundationsponsored Alpha-1 Coded Testing (ACT) Study that involves primarily family testing.

Although all of these approaches have shown an increase in the testing rate, only a few have been implemented with success in real-world medicine.

Efforts to promote the detection of Alpha-1 must continue, and this should include assessing the potential impact of direct-to-consumer genetic testing and revisiting newborn testing.

The third session included presentations on “Emerging Biomarkers in Alpha-1 Antitrypsin Deficiency and COPD.” Christine Wendt, MD, from the University of Minnesota, reviewed exciting new biomarkers that could eventually assume a significant role in COPD research, especially clinical trials investigating new therapeutic interventions.

Monica Goldklang, MD, from Columbia University, presented data on a new non-invasive imaging technique that can quantitate apoptosis and is applicable to COPD. Robert A. Sandhaus, MD, PhD, FCCP, from National Jewish Health and clinical director of the Alpha-1 Foundation, then reviewed two biomarkers that have already been successfully tested in patients with Alpha-1-related COPD. One is computer-assisted tomography of the chest to quantitate lung tissue density as a marker of lung tissue destruction, a hallmark of COPD. The other is the

serum level of desmosine and isodesmosine, two amino acids that are uniquely contained in lung elastin.

As lung elastin is destroyed in emphysema, the serum levels of these amino acids are increased and could be used as biomarkers of emphysema. The process of introducing new biomarkers as outcomes in clinical trials is complicated and involves buy-in from pharma, academia and regulatory agencies. Therefore, the COPD Biomarkers Qualification Consortium was created in 2010.

The meeting ended with an extended panel discussion, moderated by Adam Wanner, MD, scientific director of the Alpha-1 Foundation, to identify next steps for the Foundation to consider. The panel consisted of the speakers and included additional experts including Jeanine D’Armiento, MD, PhD, from Columbia University and chair of the Alpha-1 Foundation’s board, Mark Brantly, MD, from the University of Florida, and Gerard Turino, MD, from Columbia University.

The panel members made two strong recommendations: 1. The Alpha-1 and COPD Foundations should collaborate in the rural COPD initiative and firmly embed testing for Alpha-1 in the program.

2. The Alpha-1 Foundation should announce a request for research grant applications (RFA) to test the effect of alpha-1 antitrypsin boosting during an acute COPD exacerbation in Alpha-1.

Overall, the meeting met its goal of summarizing the state of the art in three important topics pertaining to the COPD National Action Plan: role of exacerbations in COPD, detection of COPD in persons with or without Alpha-1, and biomarkers of COPD. 1 ALPHA-1 FOUNDATION

Background photo: Panel members discuss the Alpha-1 Foundation’s approach to the COPD National Action Plan at the 17th Gordon L. Snider Critical Issues Workshop, in New York. November 2018. www.alpha1.org

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FRONTIERS

EARCO: European Alpha-1 Research Collaboration

T

he European Alpha-1 Research Collaboration (EARCO) is a Clinical Research Collaboration of the European Respiratory Society (ERS). EARCO establishes opportunities for collaboration among multiple stakeholders, including researchers, healthcare providers, patients, and industry. The goal is to advance understanding through clinical and scientific research and improve the quality of life for patients with Alpha-1 Antitrypsin Deficiency (Alpha-1).

Although great improvements have been made in the understanding of Alpha-1 in recent years, EARCO states that many questions remain unanswered and that the genetic condition poses several important challenges: • A large proportion (about 90 percent) of individuals affected by the disorder remain undiagnosed; therefore, they have no access to appropriate care and treatment.

• Although the vast majority of patients with severe deficiency share the same genetic disorder (ZZ genotype), the prevalence and severity of liver and respiratory disease vary noticeably. Genetic cofactors have been suspected, but the majority have yet to be identified.

• The natural history and prognosis of Alpha-1 patients is not well-known, and there is a lack of prognostic tools to support medical decisions, for example, early referral for lung transplantation.

• Most studies on Alpha-1 have focused on the ZZ population, while the impact of other genotypes, such as SZ or rare deficient variants, on outcomes is less well-known. The answers to these questions require access to large cohorts of patients and cannot be addressed adequately even at the country level.

The first objective of EARCO is the design and implementation of a prospective, international registry of Alpha-1 patients, in order to understand the natural history of the condition and investigate the impact of

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different therapies, including augmentation therapy, on the course of the condition.

The European Lung Foundation actively participated in the elaboration of the recent ERS statement on diagnosis and treatment of pulmonary disease in Alpha-1 individuals. Among the most frequent issues raised by patients were the need for a global registry, more information about comorbidities, natural history of the disease, and risk factors for progression and poor prognosis of lung disease.

Therefore, the EARCO registry is the response to this unmet need, and it will work in close collaboration with the European Reference Network (ERN) for respiratory diseases (ERN-LUNG), the ERN for hepatological diseases (ERN RARE-LIVER) and the registry of the Alpha-1 Liver Group in Europe.

The EARCO registry is modeled in part on the Alpha One International Registry (AIR) group established in 1997, which included representatives from 14 European countries and was successful in stimulating international collaborative research and organizing and developing clinical trials. However, no real-life, longitudinal data was systematically collected.

The EARCO registry will also take advantage of the existing Alpha-1 registries that have been developed at the national level in several European countries, by harmonizing the data collection and assessing the quality of the data.

Over the next three years, EARCO will set up the new European-based Alpha-1 registry and establish the roadmap for clinical and translational research in the field.

The collaboration of all stakeholders and the inclusion of patients as active participants in the development of EARCO makes it highly likely that it will generate new knowledge with direct impact on patients’ quality of life and clinical care. 1 ALPHA-1 FOUNDATION

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Objectives of EARCO • Build a network of patients, researchers and clinical experts in Alpha-1 to guide future clinical and research priorities in Europe • Create the EARCO prospective registry with a quality control system, a European Alpha-1 registry to facilitate patient recruitment for research and quality improvement initiatives across healthcare systems • Establish a consensus among patients and physicians on the main clinical and translational research priorities in the field of Alpha-1 • Support and encourage early career researchers in the field of Alpha-1 through involvement in network activities • Increase the number and quality of clinical trials performed in Alpha-1 across Europe

www.alpha1.org

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ALPHA LIFE

A generous gift matched, honoring two decades of excellence ast October, AlphaNet made a commitment to match a gift that was generously donated by the Cadwgan family to the Alpha-1 Foundation as a sign of their deepest gratitude for Robert C. Barrett’s years of service at AlphaNet. After 20 years leading AlphaNet, Barrett retired knowing that the company had contributed more than $55 million to the Foundation to support Alpha-1 research and related programs. The Cadwgan family gift, in the amount of $100,000, was given in honor of Barrett’s longstanding service.

The full gift amount of $200,000, combining the Cadwgan family’s gift and AlphaNet’s match, will continue to move the Foundation’s mission forward: to find a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1) and improve the lives of people affected by Alpha-1 worldwide.

“This contribution came about from two ideas, one was that we needed to increase our revenue at the Foundation, and it also gave a chance to interact with my sister, Carol Lavell, who is an Alpha-1 Carrier, and get her involved, even though she has made contributions in the past. Finally, it was an opportunity to thank Bob Barrett for his contributions to the Foundation, as the gift is being given in his honor,” expressed Cadwgan.

The Cadwgan family has been a generous donor of the Alpha-1 Foundation for many years, and recognized in the Founder’s Circle, with donations exceeding $1 million over the years.

The Alpha-1 Foundation takes this opportunity to express its deepest appreciation and sincere gratitude to AlphaNet and the Cadwgan family for these incredible gifts that bring us one step closer to finding a cure for Alpha-1.

Gordon E. Cadwgan, PhD, immediate past chair, Board It is through generous of Directors, Alpha-1 Foundation (left); Robert C. Barrett, contributions like former president and CEO of AlphaNet (right)

“The AlphaNet board of directors and staff felt strongly that the leadership of the Cadwgan family, in honoring Barrett’s service to Alphas, needed to be matched. It is a pleasure to further the Foundation’s mission in doing so,” said Mark Delvaux, president and CEO of AlphaNet. An Alpha himself, Gordon E. Cadwgan, PhD, is the Foundation’s immediate past chair of the board of directors and has been involved in the Alpha-1 community with his wife, Ruth, for decades.

As an Alpha-1 patient, Cadwgan feels it is his responsibility to contribute to the Foundation’s mission and programs.

these that the Alpha-1 Foundation has been able to develop a solid infrastructure to promote research and the development of new therapies for improving the quality of life for those diagnosed with Alpha-1.

Each dollar the Foundation receives brings us closer to the cure and allows us to foster collaborations with investigators throughout the United States and Europe, the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), people affected by Alpha-1, and the pharmaceutical industry, to expedite the development of improved therapies, and ultimately find a cure for Alpha-1. 1 ALPHA-1 FOUNDATION

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1-877-2-CURE-A1 (228-7321)

NOW AVAILABLE READY-TO-INFUSE

PROLASTIN C LIQUID

The #1 prescribed alpha-1 therapy for over 25 years is now available in a new, ready-to-infuse liquid formulation.

• 15-minute infusion time remains the same • Proven to effectively raise the alpha1-antitrypsin protein levels in patients with alpha1-antitrypsin deficiency, also known as alpha-1

• With PROLASTIN-C LIQUID, all your PROLASTIN DIRECT® program benefits and services remain the same

• The PROLASTIN DIRECT program gives you the confidence that ongoing personalized support is there for you when needed

Steve M. and his wife, Ellen.

Steve has been on therapy since 2007.

Visit our new website, www.prolastin.com/LIQUID, for more information and patient-friendly resources.

Important Safety Information PROLASTIN®-C LIQUID is an alpha1-proteinase inhibitor (human) (alpha1-PI) indicated for chronic augmentation and maintenance therapy in adults with clinical evidence of emphysema due to severe hereditary deficiency of alpha1-PI (alpha1-antitrypsin deficiency).

Hypersensitivity reactions, including anaphylaxis, may occur. Monitor vital signs and observe the patient carefully throughout the infusion. If hypersensitivity symptoms occur, promptly stop PROLASTIN-C LIQUID infusion and begin appropriate therapy.

Limitations of Use • The effect of augmentation therapy with any alpha1PI, including PROLASTIN-C LIQUID, on pulmonary exacerbations and on the progression of emphysema in alpha1-PI deficiency has not been conclusively demonstrated in randomized, controlled clinical trials

Because PROLASTIN-C LIQUID is made from human plasma, it may carry a risk of transmitting infectious agents, eg, viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. This also applies to unknown or emerging viruses and other pathogens.

• Clinical data demonstrating the long-term effects of chronic augmentation or maintenance therapy with PROLASTIN-C LIQUID are not available

The most common adverse reactions during PROLASTIN-C LIQUID clinical trials in >5% of subjects were diarrhea and fatigue, each of which occurred in 2 subjects (6%).

• PROLASTIN-C LIQUID is not indicated as therapy for lung disease in patients in whom severe alpha1-PI deficiency has not been established PROLASTIN-C LIQUID is contraindicated in immunoglobulin A (IgA)-deficient patients with antibodies against IgA or patients with a history of anaphylaxis or other severe systemic reaction to alpha1-PI products.

© 2018 Grifols

Please see brief summary of the full Prescribing Information for PROLASTIN-C LIQUID on adjacent page.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

All rights reserved

July 2018

BN/PRL/0618/0047(1)

PROLASTIN®-C LIQUID Alpha1-Proteinase Inhibitor (Human)

HIGHLIGHTS OF PRESCRIBING INFORMATION These highlights do not include all the information needed to use PROLASTIN®-C LIQUID safely and effectively. See full prescribing information for PROLASTIN-C LIQUID. PROLASTIN®-C LIQUID (Alpha1-Proteinase Inhibitor [Human]) Solution for Intravenous Injection Initial U.S. Approval: 1987

----------------- DOSAGE FORMS AND STRENGTHS -----------For injection: approximately 1,000 mg in a single-use vial containing 20 mL of solution for injection.

------------------------- CONTRAINDICATIONS ------------------• Immunoglobulin A (IgA) deficient patients with antibodies against IgA. • History of anaphylaxis or other severe systemic reaction to Alpha1-PI. ------------------- WARNINGS AND PRECAUTIONS -------------• Severe hypersensitivity and anaphylactic reactions may occur in IgA deficient patients with antibodies against IgA. Discontinue administration of the product and initiate appropriate emergency treatment if hypersensitivity reactions occur.

• Because PROLASTIN-C LIQUID is made from human --------------------- INDICATIONS AND USAGE ---------------plasma, it may carry a risk of transmitting infectious PROLASTIN-C LIQUID is an Alpha1-Proteinase Inhibitor agents, e.g., viruses, the variant Creutzfeldt-Jakob (Human) (Alpha1-PI) indicated for chronic augmentation disease (vCJD) agent, and, theoretically, the Creutzfeldtand maintenance therapy in adults with clinical evidence of Jakob disease (CJD) agent. emphysema due to severe hereditary deficiency of Alpha1-PI ------------------------ ADVERSE REACTIONS ----------------------(alpha1-antitrypsin deficiency).

The most common adverse reactions during PROLASTIN-C Limitations of Use LIQUID clinical trials in > 5% of subjects were diarrhea and • The effect of augmentation therapy with any Alpha1-PI, fatigue, each of which occurred in 2 subjects (6%). including PROLASTIN-C LIQUID, on pulmonary exacerbations and on the progression of emphysema To report SUSPECTED ADVERSE REACTIONS, contact in Alpha1-PI deficiency has not been conclusively Grifols Therapeutics LLC at 1-800-520-2807 or FDA at demonstrated in randomized, controlled clinical trials.

1-800-FDA-1088 or www.fda.gov/medwatch.

• Clinical data demonstrating the long-term effects of chronic augmentation or maintenance therapy with PROLASTIN-C LIQUID are not available.

• PROLASTIN-C LIQUID is not indicated as therapy for lung disease in patients in whom severe Alpha1-PI deficiency has not been established.

------------------- DOSAGE AND ADMINISTRATION -------------For intravenous use only.

• Dose: 60 mg/kg body weight intravenously once per week.

• Dose ranging studies using efficacy endpoints have not been performed with any Alpha1-PI product, including PROLASTIN-C LIQUID. • Administration: 0.08 mL/kg/min as determined by patient response and comfort.

Grifols Therapeutics LLC Research Triangle Park, NC 27709 USA 3052603 U.S. License No. 1871 Revised: 08/2018

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1-877-2-CURE-A1 (228-7321)

By Joan Garry

N

early 30 years ago, my husband Oliver and I arrived in Boston, Massachusetts.

We came from Ireland with our oldest child, Alan. Our daughter, Joanna, was born in 1994, followed by our youngest daughter, Catriona, in 1996.

Born at Tufts Medical Center and weighing just over 5 pounds, Catriona was a tiny girl with no baby fat at all. Her color was pasty; she had no interest in eating and was just clinging at the bottom of the growth chart.

It took a few months to establish a definite diagnosis. Initially, the doctors said she had “neonatal hepatitis of unknown etiology,� but further testing and final results indicated that she had Alpha-1 Antitrypsin Deficiency (Alpha-1) and that she did not just have low levels of alpha-1 antitrypsin, but almost none at all.

From the very beginning, Catriona was severely affected. She grew very slowly, her liver was very enlarged, and her liver function tests kept deteriorating. She was a tiny girl with a swollen belly and stick-like arms and legs. Her entire first summer was spent in the hospital undergoing a series of medical complications. The fluid in

her belly was drained many times, but it always reaccumulated, and it became infected with dangerous bacteria.

When Catriona finally left the hospital, she had a feeding tube surgically inserted into her gastrointestinal tract (g-tube), a central intravenous line surgically inserted into her chest, and she was on a complex regimen of medications.

At five months old, she had infant cirrhosis and was listed for a life-saving liver transplant.

After two long years of g-tube feedings and just before her third birthday, Catriona reached the 20-pound mark recommended by the transplant team and we received the call that a liver was available for her.

ALPHA-1 KIDS

A mother’s perspective

Catriona went into surgery Initially, her recovery went well. However, just two weeks post-transplant, she had two major bleeds from esophageal varices (blocked veins in the esophagus caused by her liver disease), something that is more likely seen in adult-alcoholics with years of cirrhotic liver disease. They were frightening episodes to witness, but thankfully she pulled through and got better.

Being a normal 22 year-old is very important to Catriona. Traveling is something that she loves to do, and she has learned how to take control of her own health. From scheduling her medications to being prepared on her trips, she has navigated how to live her best life.

Catriona traveling and enjoying Switzerland.

www.alpha1.org

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ALPHA-1 KIDS

She continued to improve. Her g-tube and central line were removed, and her varices resolved. Oliver and I were confident that the worst was over, and we resumed our plans to move back home to Ireland.

The kids were excited about the big move.

But after some time, we were back on the medical rollercoaster ride. Catriona’s health took a turn for the worse at 12 years old. Her weakened immune system and the side effects of the antirejection drugs contributed to her failing health. Her blood pressure rose to dangerous levels, her weight plummeted, she developed pseudomonas infections in her lungs, and her failing kidneys were the cause of hypertensive seizures.

Catriona Garry with mother, Joan Garry

She was listed for a kidney transplant Fortunately, her father was a match and he donated one of his kidneys to save our daughter’s life.

Catriona is now 22 and is studying communications at Lasell College, in Newton, Massachusetts. She strives to live a normal life. She is a strong-willed young lady, determined to face each day with hope and live life to the fullest.

The Garrys have become very involved in supporting the Alpha-1 community. Living in the Boston area, they have enjoyed participating in the annual Escape to the Cape Bike Trek and the annual Celtic Connection celebrations.

Alpha-1 has certainly impacted our lives and turned our plans upside down. Complications from this genetic condition nearly took our child’s life. I spent most of Catriona’s childhood in the hospital with her, and Oliver worked long hard hours to keep us afloat, while our plan to return to Ireland has been shelved.

Despite the commotion Alpha-1 has caused in our lives, we try to look at the glass half-full. We are very thankful to be living near Boston, a great city that offers excellent health care and we are very appreciative of the generosity of organ donor families who selflessly give, under tragic circumstances, so that others may receive the gift of life.

We are also very grateful for the commitment of the Alpha-1 Foundation to finding a cure for people living with Alpha-1. I have shared our story to clinicians and researchers to help inspire them, and to thank them for their everyday work.

As the mother of an Alpha-1 child, it is wonderful to think that, with new medical advances and emerging technologies, serious and hereditary genetic conditions, like Alpha-1, will be a thing of the past. 1 ALPHA-1 FOUNDATION

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1-877-2-CURE-A1 (228-7321)

The

A

lph

a

of T omo r r o w

28th Annual Alpha-1 National Conference JUNE 21-23, 2019 | ORLANDO, FL

PLATINUM SPONSORS: AlphaNet | CSL Behring | Grifols | Takeda GOLD SPONSOR: Eversana

T H U R S D A Y , J u n e 2 0 th 3:00 pm – 8:00 pm

CSL Behring’s 12th Annual Walk for Breath Celebration

F R I D A Y , J u n e 2 1 st 8:30 am – 10:00 am

Breakfast Sponsored by Takeda

10:00 am – 12:30 pm Track 1: Conference Orientation and Alpha 101 - Lung and Liver 9:30 am – 12:45 pm

Track 2: The Path to Finding a Cure - Open Scientific Sessions

1:00 pm – 2:00 pm

Luncheon Sponsored by Grifols

2:00 pm – 5:30 pm

Meet the Experts, Building Friends for a Cure Training & Networking

4:00 pm – 5:00 pm

Repeat Sessions: Alpha 101 - Lung and Liver

6:30 pm – 9:00 pm

Friday Evening Reception Breakfast

8:30 am – 12:00 pm

Keynote Presentation and General Sessions including a Research Panel

12:00 pm – 1:00 pm

Meet the Speakers

1:00 pm – 2:00 pm

Lunch

2:00 pm – 5:10 pm

Four Afternoon Tracks: Where We Are Going With the Liver, Other Diseases Concurrent with Alpha-1, How to Be Your Own Advocate, Transplantation, Genetics & the Importance of Family Testing, Access & Reimbursement: Insurance Issues, The Joys & Challenges of Parenting an Alpha-1 Child & more!

7:00 pm - 9:30 pm

Dinner & Awards

8:00 am – 9:30 am

Breakfast

8:30 am – 9:30 am

Memorial Service

CONFE

RENCE-A

A T-

GL

S U N D A Y , J u n e 2 3 rd

CONFERENCE REGISTRATION

KIDS & TEENAGERS

CONFERENCE LOCATION

NURSING CEU CREDITS

HOTEL RESERVATIONS

OXYGEN TRAVEL FUND

Online at a1f.org/alpha1conference or by mail with the form enclosed.

Deadline: May 15th Omni Orlando Resort at ChampionsGate 1500 Masters Boulevard ChampionsGate, FL 33896 1 (888) 444-OMNI

Online: a1f.org/orlando2019 By phone: 1-888-444-OMNI Room Rate: $149 per night plus tax Rate Deadline: May 28th Group Code: Alpha-1 Foundation National Conference

AGENDA & FAQs

ANC

7:30 am – 8:15 am

E

S A T U R D A Y , J u n e 2 2 nd

The Alpha-1 Kids program sponsors activities during the Saturday sessions for children and teens.

AlphaNet is pleased to offer a free educational program for nurses on Saturday from 2 to 4 pm. Three (3) CEU credits will be offered for nurses participating in this program.

The Oxygen Travel Fund provides oxygen and equipment for Alphas in financial need to travel to Alpha-1 educational events. To apply, please call (877) 228 - 7321 ext. 251 with at least 30 days notice.

The conference at a glance is subject to change. For a detailed Conference Agenda and Things You Need To Know, visit a1f.org/alpha1conference.

28th Annual Alpha-1 National Conference The Alpha of Tomorrow ORLANDO, FL • June 21 - 23, 2019

Registration Deadline: Monday, May 15, 2019

Patient, Family Member & Caregiver Registration First Name

Middle Name

Last Name

Title

Organization, if applicable

Address City

State/Province

Zip/Postal Code

E-mail

Phone number

Check all that apply:

First-time Attendee Caregiver

Special Dietary Need:

None

Support Group Member - SG Name: ________________________________________ Medical Professional - Type: ______________________________________________ Other: _________________________________________________________________

Vegan

Vegetarian

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Spouse & Guest Information Guest 1: First Name

Last Name

All that apply: Child/Teen:

First-time Attendee Child or

E-mail

Spouse

Caregiver

Teen | Age: _____ | Sex:

Female or

Relation: __________ | Dietary Need:

None

Vegan

Vegetarian

Male | Attending the Kids/Teen Room on Saturday, June 22nd:

Yes

Gluten-free No

Guest 2: First Name

Last Name

All that apply: Child/Teen:

First-time Attendee Child or

E-mail

Spouse

Caregiver

Teen | Age: _____ | Sex:

Female or

Relation: __________ | Dietary Need:

None

Vegan

Vegetarian

Male | Attending the Kids/Teen Room on Saturday, June 22 : nd

Yes

Gluten-free No

Guest 3: First Name

Last Name

All that apply: Child/Teen:

First-time Attendee Child or

E-mail

Spouse

Caregiver

Teen | Age: _____ | Sex:

Female or

Please check the sessions & meals you and/or your guests will be attending: Guest 1

Guest 2

Type:

Vegan

Vegetarian Yes

Gluten-free No

Visa

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AMEX

Discover

Guest 3

CSL Behring Walk for Breath Friday Takeda Breakfast Friday Grifols Luncheon Friday Evening Reception Saturday Breakfast Saturday Lunch Saturday Dinner Sunday Breakfast

Registration Fees

None

Male | Attending the Kids/Teen Room on Saturday, June 22nd:

Payment Information

Meal Information

You

Relation: __________ | Dietary Need:

Name as it appears on card Card Number Authorized Amount $

#

Total

Attendee

$ 150 x ______ = $__________

Each Spouse/Guest

$ 135 x ______ = $__________

Children 12 & under $ 110 x ______ = $__________ One-day Registration $ 135 x ______ = $__________ Total to be charged:

CVV # Expiration Date

Signature Industry/Exhibitors: If you are interested in registering as an exhibitor, researcher, and/or industry professional, or you are employed by an industry company, including MSLs and healthcare providers, please contact Alexis Artiles Ojeda at aartiles@alpha1.org.

$__________

Registration Form Page 1 of 2. Please complete both sides. Register online: a1f.org/alpha1conference

Check

28th Annual Alpha-1 National Conference The Alpha of Tomorrow ORLANDO, FL • June 21 - 23, 2019

Registration Deadline: Monday, May 15, 2019

Patient, Family Member & Caregiver Registration Travel Information I/We will be driving to the conference.

I/We will be flying to the conference and plan to use the airport shuttle. If yes, arrival/departure information is required.

Arrival Date: __________________________________________ Arrival Time: __________________________________________ Arrival Airline & Flight #: ______________________________________________________________________________________ Departure Date: _______________________________________ Departure Time: _______________________________________ Departure Airline & Flight #: ___________________________________________________________________________________

Attendee CHECKLIST Reserve your discounted hotel room Online: 1. Reserve a1f.org/orlando2019

via phone: 2. Reserve Call 1-800-THE-OMNI

Group code: Alpha-1 Foundation National Conference

Venue: Omni Orlando Resort at ChampionsGate Room Rate: $149 per night plus tax. This group rate will be honored up to three (3) days before and three (3) days after the official meeting dates based on availability at the hotel when the reservation is made.

Discounted Rate Deadline: Reservations must be made on or before Tuesday, May 28, 2019 in order to be eligible for the group rate.

Book travel arrangements Airport: Orlando International Airport (MCO)

Parking: Self-parking at the hotel/venue will be available for a discounted rate of $10 per day.

Register to attend the conference

more information Fax this completed 1. For and to register online: 2. form to: a1f.org/alpha1conference

ATTN: Kim Caraballo at (305) 503-4629

this completed form 3. Mail with payment to: Alpha-1 Foundation ATTN: Kim Caraballo 3300 Ponce de Leon Blvd Coral Gables, FL 33134

Be advised that in accordance with the provisions of the Physician Payments Sunshine Act, 42 U.S.C. 1320a-h and its implementing regulations (“PPSA”) and Alpha-1 Foundation’s contractual obligations related thereto, Alpha-1 Foundation may disclose to third parties, or otherwise make public, any payment or other transfer of value (as such term is defined in the PPSA) it makes to any physician or teaching hospital.

Registration Form Page 2 of 2. Please complete both sides. Register online: a1f.org/alpha1conference

T

he Alpha-1 National Conference is the largest annual gathering of patients diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1), their families, industry representatives, and healthcare professionals such as respiratory therapists, nurse practitioners, physicians, and nurses, in the world. It is a three-day event with educational programs given by experts and leaders in the field of Alpha-1 that provides attendees and exhibitors the opportunity to network and increase their understanding of Alpha-1 and related topics.

This year, the 28th Annual Alpha-1 National Conference is taking place in Orlando, Florida, from June 21-23, and the Alpha-1 Foundation is introducing a new scientific track, “The path to finding a cure,” on Friday, June 21.

Topics to be discussed include: • Alpha-1 – the disease and its detection • Alpha-1 Foundation research programs • The Foundation’s venture philanthropy activities • New therapies for lung and liver disease • Obstacles to finding a cure Speakers and researchers include: • Mark Brantly, MD, professor and chief, Division of Pulmonary, Critical Care & Sleep Medicine, vice chairman of research, Department of

Medicine, professor of medicine, molecular genetics and microbiology and Alpha-1 Foundation research professor at the University of Florida • Adam Wanner, MD, scientific director of the Alpha-1 Foundation • Jean-Marc Quach, president & CEO of The Alpha-1 Project (TAP) • Igor Gonda, PhD, biotechnology expert and TAP board member • Jeanine D’Armiento, MD, PhD, chair of the board of directors of the Alpha-1 Foundation, associate professor of medicine in anesthesiology, director of the Center for Molecular Pulmonary Disease in Anesthesiology and Physiology and Cellular Biophysics, and director of the Center for LAM and Rare Lung Disease “We are excited to have the new scientific track at the Alpha-1 National Conference as an initial step in our vision to bring all components of our community together. Our patients, scientists, and medical experts will have the opportunity to interact at a single site and gain knowledge from each other,” said D’Armiento.

“We are making progress in our quest for finding new treatments for Alpha-1. We believe that the presentations will inform patients and potential donors and investors about the Foundation’s research activities and partnerships with industry,” expressed Wanner.

The Annual Alpha-1 National Conference provides a unique opportunity to learn about Alpha-1, and to get the latest information, directly from the source, on this genetic condition. 1 ALPHA-1 FOUNDATION To learn more about the 28th Annual Alpha-1 National Conference and to register, please visit a1f.org/alpha1conference.

IN YOUR INTEREST

“The path to finding a cure,” new scientific track at National Conference

Adam Wanner, MD, scientific director of the Alpha-1 Foundation

www.alpha1.org

15

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16

1.877.2.CURE.A1 (228.7321)

Alpha-1 awareness

T

he month of November has been marked by the Alpha-1 Foundation as a month to raise awareness about Alpha-1 Antitrypsin Deficiency (Alpha-1), through several activities designed to increase knowledge and spread awareness about this condition that affects at least 100,000 people in the United States and several other thousands worldwide.

During Alpha-1 Awareness Month, those affected by Alpha-1 share their stories, their challenges and successes of living with the condition, and the importance of Alpha-1 research and early detection.

Alphas, along with their families and friends from all over the United States, joined together for the annual Alpha-1 Foundation Virtual Walk. For 30 days, in 36 states, friends and family supported one strong mission to find a cure. From near and far, 68 teams and 40 solo walkers added up to 785 donors and participants who walked, ran, or found their unique way to raise awareness and dollars towards a cure.

The Foundation is proud of all the Virtual Walk participants that made their mark in their communities.

This year was the most active and diverse to date as support groups, teams, and families met at plasma centers, state capitols, shopping malls, churches and even theme parks. Football games were played, rocks were painted, and some medals were won at 5K and 10K events.

Whether in memory or honor of loved ones, together purple shirts were worn, and the awareness was spread.

Many offices joined in for this year’s activities. Dedicated employees from the offices of the Alpha-1 Foundation, Accredo, CSL Behring, Eversana and Grifols Plasma Centers laced up their sneakers for Alphas worldwide.

Together, the mission is stronger, and the community raised $76,000 towards the cure!

Alphas all over the United States asked their governors and mayors to declare the month of November Alpha-1 Awareness Month. The community exceeded all goals and obtained 56 proclamations from around the nation and told their stories to the state and local representatives.

The proclamations will be proudly displayed at the 28th Annual Alpha-1 National Conference in June. www.alpha1.org

Alpha-1 Foundation staff wearing stripes for Rare Disease Day

IN YOUR INTEREST

Near and far, each step brings us closer to a cure

Rare Disease Day

On Feb. 28, 2019, the Alpha-1 Foundation proudly wore its stripes to support the National Institutes of Health (NIH) sponsored Rare Disease Day (RDD) for their “Show Your Stripes” campaign, along with patient organizations, politicians, caregivers, medical professionals, researchers and industry – together to raise awareness of rare diseases, including Alpha-1.

Throughout the entire month of February, the Foundation promoted awareness in conjunction with our membership organization NORD - the National Organization for Rare Disorders and the NIH. The theme for RDD 2019 was: “Bridging health and social care,” focusing on bridging the gaps in the coordination between medical, social and support services in order to tackle the challenges that people living with a rare disease and their families face every day, such as the Alpha-1 community.

The big news at this year’s Rare Disease Day was the NIH launch of an online “how-to” tool for patient advocacy 17

IN YOUR INTEREST

Anita and Keith Sellers, from Arkansas, start their fundraising efforts a year in advance with “Change for a Cure,” which allowed them to collect $1,000 in loose change. They raised and contributed to their grand total of $7,000, from friends and family that sponsored Anita’s 150-mile walk, on behalf of her husband, Keith, who was diagnosed with Alpha-1 in 2002.

“We are in this fight together, we all need to raise Keith Sellers with grandchildren awareness and funds for research. Anything we do, no matter how small it seems, adds up in the big picture. Our friends and family will donate, we just need to ask,” Anita said.

organizations to bring their registries in-house. The NIH, National Center for Advancing Translational Sciences (NCATS) presented on their new Rare Diseases Registry Program (RaDaR), which was created to provide accessible advice on setting up and maintaining good-quality registries for rare diseases to stimulate therapeutic development. In the next phase, the RaDaR’s website will add content about how to transform a contact registry into a patient registry with clinical data.

Alphas were represented at the Rare Disease Legislative Advocates (RDLA) events throughout Rare Disease Week on Capitol Hill, from Feb. 24-28. Over 500 rare disease patient advocates joined in Washington, DC, and met with their senators, representatives and Congressional staffers to discuss key legislation, policies and the Rare Disease Congressional Caucus. Our Alpha-1 patient representatives learned best practices for successful advocacy, federal legislation and policies that affect the rare disease community.

The social media outlets were flooded with Alphas wearing stripes to raise awareness and showing their support of Rare Disease Day on Facebook, Instagram and Twitter. The Foundation participated in the Feb. 22 Twitter chat hosted by the NIH in coordination with NCATS and the NIH Clinical Center. The hour long discussion joined organizations and people from 44 states and 59 countries to spread awareness on their rare disease and provide patients a platform to share what tools and resources they use to connect with other members in the rare disease community. Over 1,200 participants generated 4,164 tweets in just 60 minutes!

Every day is a day to raise awareness on rare diseases, and we are grateful for our partnerships that allow your voices to be heard. Visit our website to learn more

about becoming an Alpha-1 Advocate or to start your Virtual Walk team for Alpha-1 Awareness Month this November.

Thank you to AlphaNet, CSL Behring, Eversana, Grifols, and Takeda for their outstanding support of the FOUNDATION Virtual Walk and our awareness campaigns. 1 ALPHA-1

Jeanine D’Armiento, MD, PhD, chair of the board of directors; DC Young, Alpha-1 Ambassador; Miriam O’Day, president & CEO, Alpha-1 Foundation

This year’s Rare Disease Day slogan was “Show your stripes.” The zebra is the official symbol of rare diseases in the United States and is noted for its black and white stripes, which are central to its uniqueness.

While each of the more than 7,000 rare diseases is unique, there are many things in common that unite the rare disease community together to raise awareness. “Zebra” is the American medical slang for arriving at an exotic medical diagnosis when a more commonplace explanation is more likely.

18

1-877-2-CURE-A1 (228-7321)

he Alpha-1 Foundation lost a dear friend and leader, James (Jim) Quill on Sunday, Jan. 13, 2019, in Bluffton, South Carolina. He was 67.

Quill was on the Alpha-1 Foundation Board of Directors since 2012 and recently served as the vice chair and treasurer. He was a dedicated AlphaNet coordinator for 10 years and retired as the general manager for AlphaNet, in 2018. He was loved and respected by his colleagues and all the Alphas he served. He touched the lives of so many and was a cherished friend to all who knew him.

Originally from Levittown, Pennsylvania, Quill spent 35 years giving the gift of education to elementary school students as a classroom teacher, reading specialist, and a principal. He was diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) in the late 1980s and received a bilateral lung transplant in May 2006. Following his transplant, he began his new career as an AlphaNet coordinator and helped people learn more about their health and how to live with Alpha-1. Alpha-1 affected Jim’s whole family, and he offered a steady reassurance to all he interacted with.

“Jim was a dedicated, selfless Alpha. He had a gentle soul and as a board member was a strong voice for the needs of Alphas. I will forever be grateful to Jim for his service to the community and guidance to me personally while serving on the board of the Alpha-1 Foundation. He was a valuable member of our community who provided sound advice to all of us. He will be greatly missed,” said Jeanine D’Armiento, MD, PhD, chair of the Alpha-1 Foundation board of directors.

A Foundation event was not complete without Jim and his family there to support it. He was dedicated to the mission of finding a cure for Alpha-1 and worked side by side with the Foundation to inspire Alphas. He recently served on the Foundation’s Educational Materials Working Group, the Budget, Program & Nominating Committee, and the Audit, Finance, and Compensation Committee. He was an active volunteer for the Gift of Life Program and regularly attended Alpha-1 Support Groups and Education Days.

Quill received the Peter Smith Achievement Award in 2015, named for the first Alpha to distribute a version of Alpha-1 news intended to keep Alphas informed. At the 2018 Alpha-1 National Conference, last summer, Quill was awarded the Inspirational Award for his many years of dedicated service, vision as a

www.alpha1.org

leader, Alpha-1 advocate, and fundraiser for the Alpha-1 community. He was recognized upon his retirement from AlphaNet at the 2018 Celebration of Life, the Foundation’s signature fundraising event.

“All at AlphaNet are saddened by the passing of Jim Quill. Over the last decade, since Jim joined AlphaNet, he was an AlphaNet coordinator and eventually the general manager of the AlphaNet program. In each role he drew on his experience as a teacher and school principal to provide education and guidance to those he served,” said Robert A. Sandhaus, MD, PhD, FCCP, medical director of AlphaNet. “Ever soft-spoken and even-tempered, even when those around him were not, he provided calm direction and wisdom to all he touched, including the many coordinators and several thousand patients with Alpha-1 under his supervision. It would be hard to imagine AlphaNet without his guiding hand, except that he was able to surround himself with highly qualified protégés that together can step up to carry on his work,” he added.

Jim Quill is survived by his loving wife Marge, of 45 years; his sons and daughters-in-law Donovan, Holly, Andrew and Holly; his five grandchildren, as well as, nieces and nephews.

The Alpha-1 Foundation is grateful to Jim for his contributions to the Alpha-1 community. He will be FOUNDATION deeply missed. 1 ALPHA-1

ALPHA LIFE

Honoring Jim Quill

19

ASK THE ALPHA DOC

How is panniculitis diagnosed?

P

anniculitis requires a biopsy of the skin to make doses than those the diagnosis. The classic lesion is painful, used in treating discolored (usually dark purple), raised, and Alpha-1-related eventually oozing and healing with a scar – but lung disease. classic lesions are the exception rather than the rule. Some Q. What individuals have nodules under the skin, particularly in explanation is fatty areas of the body that come and go. These sometimes there for low have rather minor skin redness. Although, a deep biopsy levels of alpha-1 of the skin and underlying fat is relatively easy to do, the antitrypsin if only reason to biopsy is if an Alpha, not on therapy, might you are MM want to consider starting. Inflammation and vasculitis (not an Alpha)? of the skin with necrosis under the microscope can A: The answer Charlie Strange, MD, medical definitively make the diagnosis. depends on how director of AlphaNet Q. W hat are the types of panniculitis associated with low a level one Alpha-1 Antitrypsin Deficiency (Alpha-1)? is talking about.

If the level is slightly below the normal range, this A: P anniculitis is a term that means “inflammation of fat.” can be just a normal level for an individual with There are many diseases that can do this, including some very common diseases, such as diabetes or absolutely no infection/inflammation going on. If pancreatitis. Panniculitis in Alpha-1 occurs almost the level is significantly below the normal range, one might consider rechecking the Alpha-1 genotype exclusively in ZZ individuals but has rarely been seen with more sophisticated testing. in other rare deficiency genotypes.

Q. Can an Alpha donate plasma if Q. W hat is the treatment for they are not on augmentation panniculitis? therapy or asymptomatic?

A: P anniculitis is not exclusively caused by Alpha-1. If panniculitis A: Technically, in the U.S., the national regulating body has stated is found in someone not known that alpha-1 antitrypsin deficient to have Alpha-1, the usual individuals can give blood and can first treatment is steroids, be plasma donors, but the final usually by mouth rather than a steroid cream. Sometimes other decision is usually left to the actual immunosuppressive drugs are donation center. Some Alphas have tried. Likely, all people diagnosed been turned down as plasma donors with panniculitis should be tested because of their deficient levels of for Alpha-1, although this is alpha-1 antitrypsin since it is possible Robert A. Sandhaus, MD, not in the guidelines. If a severe the donated plasma could be used PhD, FCCP, clinical director deficiency is found, the primary to make augmentation therapy, and of the Alpha-1 Foundation treatment is augmentation therapy, there would only be low levels of Z FOUNDATION and is sometimes given at higher protein in that donated unit. 1 ALPHA-1 Panniculitis is an inflammation of the panniculus, the layer of fatty and fibrous tissue just beneath the outer layers of our skin. This layer of the skin looks like a honeycomb, with globules of fat separated by walls, or septae.

Like most medical conditions, panniculitis can have many underlying causes. Alpha-1 is one of those causes. There are many other causes, including diseases that involve widespread inflammation of the body, such as lupus (systemic lupus erythematosus) and rheumatoid arthritis, and reactions to some drugs, including corticosteroids.

20

1-877-2-CURE-A1 (228-7321)

Explore, Engage, and Experience the Alpha-1 Community that CareZ

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Explore helpful educational resources • Living With Alpha-1 brochure • My Steps for Healthy Living program

Engage meaningfully with other Alphas • Peer-to-peer engagement with AlphaNet Coordinators • Community building through the Alpha-1 Foundation • Walk for Breath Celebration

Experience support when you need it most • Experienced CareZ Care Coordinators to help you navigate access • Zemaira® Signature SavingsSM* offers 12 months of renewable co-pay assistance • CSL Behring AssuranceSM* provides free Zemaira therapy in the event of a loss of or lapse in commercial insurance coverage

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Important Safety Information Zemaira® is indicated to raise the plasma level of alpha1-proteinase inhibitor (A1-PI) in patients with A1-PI deficiency and related emphysema. The effect of this raised level on the frequency of pulmonary exacerbations and the progression of emphysema have not been established in clinical trials.

In clinical studies, the following adverse reactions were reported in at least 5% of subjects receiving Zemaira: headache, sinusitis, upper respiratory infection, bronchitis, fatigue, increased cough, fever, injection-site bleeding, nasal symptoms, sore throat, and swelled blood vessels.

Please see full Important Safety Information and brief summary of prescribing information for Zemaira on the following page.

Zemaira is manufactured and distributed by CSL Behring LLC. Zemaira® and CareZ® are registered trademarks of CSL Behring LLC. Zemaira® Signature SavingsSM and CSL Behring AssuranceSM are service marks of CSL Behring LLC. ©2017 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA www.CSLBehring-us.com www.Zemaira.com ZMR-0101-OCT17

Important Safety Information ZemairaÂŽ is indicated to raise the plasma level of alpha1-proteinase inhibitor (A1-PI) in patients with A1-PI deficiency and related emphysema. The effect of this raised level on the frequency of pulmonary exacerbations and the progression of emphysema have not been established in clinical trials.

Zemaira may not be suitable for everyone; for example, people with known hypersensitivity to components used to make Zemaira, those with a history of anaphylaxis or severe systemic response to A1-PI products, and those with certain IgA deficiencies. If you think any of these may apply to you, ask your doctor.

Early signs of hypersensitivity reactions to Zemaira include hives, rash, tightness of the chest, unusual breathing difficulty, wheezing, and feeling faint. Immediately discontinue use and consult with physician if such symptoms occur.

In clinical studies, the following adverse reactions were reported in at least 5% of subjects receiving Zemaira: headache, sinusitis, upper respiratory infection, bronchitis, fatigue, increased cough, fever, injection-site bleeding, nasal symptoms, sore throat, and swelled blood vessels.

Because Zemaira is made from human blood, the risk of transmitting infectious agents, including viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be completely eliminated.

Please see brief summary of prescribing information for Zemaira below.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Based on September 2015 revision.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

R

ic Logsdon has lived in the Louisville, Kentucky area for most of his life. He was diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) at age 30, and this is how it happened.

Logsdon experienced symptoms like wheezing and difficulty breathing since childhood and was diagnosed with asthma when he was 7 years old. He was later diagnosed with bronchiectasis at the age of 17, and with emphysema, in his early 20s. Even with all of these diagnoses, he was never tested for Alpha-1. Although he never smoked, his doctor came to the conclusion that his emphysema was due to smoking.

It was upon his father’s passing in 1999 that the pathology report revealed he had Alpha-1. Up until then, they were unaware of this rare and genetic condition. Although it was recommended that the whole family be tested for Alpha-1, Logsdon waited over a year to proceed with testing.

Logsdon was diagnosed alpha-1 antitrypsin deficient in 2001.

“After my father passed away, his last great gift was the knowledge of having Alpha-1 in our family,” said Logsdon while explaining that being the youngest of eight children, he is one of three severely deficient Alphas (ZZ). He has five siblings that are Carriers (MZ), and his mother is also a Carrier.

In the Alpha-1 community, each situation is similar and unique at the same time. On average, it takes seven years for an Alpha to get diagnosed, and yet each individual has their own set of circumstances, as well as their Alpha-1 journey. After his diagnosis, Logsdon began to educate himself about Alpha-1 and get involved in the Alpha-1 community.

“After my diagnosis, I went into denial for about two years and then it got to a point where I could not do my job as a surveyor without an assistant, until I finally broke down. I needed to know more, so I started going to education days.

I went to every education day and support group meeting within 150-200-mile radius from home,” he explained.

Logsdon started weekly augmentation therapy infusions in 2003 and a few years later, in 2007, while at the Alpha-1 National Conference, he made contact with a representative from the plasma industry who offered www.alpha1.org

him a position as a patient support specialist. In 2014, Logsdon left that company and joined AlphaNet as a coordinator.

“I have been on augmentation therapy for several years and I truly believe that living right, staying fit, and communicating with other fellow Alphas, has saved my life,” he explains.

He also found engaging in advocacy very rewarding. In 2006, he traveled to Washington, DC, to speak with members of Congress and leaders on Capitol Hill about the important bills affecting “access to care” that ultimately affect Alphas and their families, including the Genetic Information Nondiscrimination Act (GINA).

“I tell everybody that has an opportunity to go to Washington, DC, that they should go to preach Alpha-1 and walk the halls. We have congressmen and senators that are not aware of this condition, and without your voice, they are not going to represent Alphas when making important decisions,” Logsdon said as he encouraged Alphas to get involved.

Logsdon has been working to educate physicians and their staff, as well as working with Alpha-1 patients to get access to care for more than 10 years.

Currently, as an AlphaNet coordinator, Logsdon continues to spread awareness of Alpha-1 and use his motto: “I have Alpha-1, but Alpha-1 does not have me.”

ALPHA-1 COMMUNITY

Ric Logsdon: “I have Alpha-1 but Alpha-1 does not have me”

“The amount of support and resources we have as a community is mind-blowing; between the Foundation, AlphaNet and its disease management program, there is so much to help people along their journey,” he explained.

Being a pastor of a small church in Jeffersontown, Kentucky, one of Logsdon’s greatest joys is helping people find their way in life, whether it is in the church or the world of Alpha-1.

“One of the things I love about being an AlphaNet coordinator is that I gain encouragement from every Alpha that I talk to. I have met some truly remarkable people and heard amazing stories during the past several years,” he concluded. 1 ALPHA-1 FOUNDATION To learn more about AlphaNet and the network of coordinators, please visit their website at www.alphanet.org.

23

ALPHA-1 COMMUNITY

ALPHAS

in the heartland Midwest Alphas support group Kansas City, MO Julie Swanson was diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) in 1988 and founded the Midwest Alphas support group three years later in 1991.

Swanson is well-known in the Alpha-1 community for her activism and support group leadership for the past 30 years. She served on the board of the Alpha-1 Association, which merged into the Alpha-1 Foundation in 2014, and was Association president for three terms.

Swanson has led the Midwest Alphas support group - one of the earliest - for part of its 28 years. The late Richard “Dick” Bueker led the group for 15 years, and during that time, Swanson was the treasurer. In 2012, Bueker stepped down and Swanson took over the leadership of the group. Her mother, Peggy Paul, has been the secretary since its inception and is now her co-leader.

“We try to cover all topics, so Alphas have an interest and come to the meetings; from pulmonary rehabilitation to how to write a will and navigating insurance,” Swanson said.

The Midwest Alphas support group continues to meet quarterly around the Kansas-Missouri border. The main focus of the group is to provide support and education to Alphas and their families.

“Alphas need to educate themselves, so they know what to ask their doctor. A good attitude is also important because it makes all the difference,” she concluded.

Greater Saint Louis Area Alpha-1 support group Saint Louis, MO For Ryan Lestina and his wife, Siobhan, their Alpha-1 journey began nine years ago. Although Siobhan was diagnosed with asthma at a young age, her physicians in Colorado were convinced that her asthma and chronic obstructive pulmonary disease (COPD) were due to smoking, but she was in her 30s and she was not a heavy smoker.

Then, they moved to Wyoming. Her new physician tested her for Alpha-1 and the test results came back positive. Their two children were tested and are both Carriers.

“We were quick to learn that although there are many pulmonologists, not enough are aware of Alpha-1,” said Lestina as he explained that he does not consider himself a caregiver, but a 24

1-877-2-CURE-A1 (228-7321)

care advocate. “We have come a long way in the past nine years, constantly learning about the condition, treatments and the research underway that will eventually lead to a cure.”

The Lestinas know they are not alone in this journey. Their Alpha-1 family grows every year and has been a constant source of support, education, laughter and kindness. They now live in St. Louis, Missouri, at a lower altitude and closer to family and medical facilities. Lestina leads the Greater St. Louis Area Alpha-1 support group and gives back as he provides support and guidance to Alphas in the area.

“With so many people from the Alpha community helping my family and I over the years, I am eager to give back and help others and their families. I’m honored to lead the newly formed Greater St. Louis Alphas support group,” he added.

The Greater St. Louis Area Alpha-1 support group recently held its very first meeting. Welcome to the Alpha-1 family!

Southwest Missouri Alphas support group Joplin, MO Karen Boyd is the support group leader of the Southwest Missouri Alphas, and although an initial blood level test for alpha-1 antitrypsin came back negative, she was referred to a pulmonologist who tested her phenotype. In June 2016 she was diagnosed as an Alpha-1 Carrier.

“My goal for the Southwest Missouri Alphas support group is to offer support and education to patients, their families, and caregivers, and to provide resources and empowerment with their diagnosis. Raising awareness and education in our community, and to local medical professionals, is also my goal for our group,” said Boyd as she shared her vision.

“I look forward to meeting you along with your family and caregivers at our meetings. Questions and suggestions are always welcome,” she concluded. 1 ALPHA-1 FOUNDATION

We are seeking support group leaders in: Alaska, Delaware, Hawaii, Mississippi, Montana, New Hampshire, New Jersey, New Mexico, North Carolina, North Dakota, Rhode Island, and Vermont.

If you are interested, please contact Barbee Bennington, support group and program coordinator, at (877) 228-7321 ext. 227, or via email at bbennington@alpha1.org.

www.alpha1.org

25

th International Research Conference on Alpha-1 Antitrypsin th Alpha-1 Global Patient Congress

4 7

3-6 April

2019 DUBROVNIK, CROATIA

A special thank you to the Global Advisory Committee:

Patient leaders, physicians, and scientists from around the world are coming together to discuss the latest developments in patient advocacy, clinical care, and Alpha-1 research. With a rich history of international collaboration, the Foundation is committed to Alphas worldwide and is proud to host representatives from 32 countries. The commitment to the mission drives the Alpha-1 Global program each day to increase awareness, detection, and access to care for Alphas around the world.

Jeanine D’Armiento, MD, PhD, Alpha-1 Foundation Chair, USA Robert Sandhaus, MD, PhD, FCCP, Alpha-1 Foundation Medical Director, USA Robert Stockley, MD, Director Lung Immuno Biochemical Research Laboratory, UK Adam Wanner, MD, Alpha-1 Foundation Scientific Director, USA

And the Global Conference Planning Committee: Cristina Barbiero, National Association Alfa-1 AT Italy, EU Advocacy Working Group, Italy Jim Clarke, Alpha-1 Association New Zealand Co-Leader Alpha-1 Support Group, New Zealand Shane Fitch, Lovexair Foundation - President, Spain Elena Goyanes, Alpha-1 Association Spain, EU Advocacy Working Group, Spain Karen O’Hara, Alpha-1 UK Support Group - ERN - Lung, EARCO, UK Carlos Cambon, Alpha-1 Association Argentina, Argentina Martina Cambon, Alpha-1 Association Argentina, Argentina Frank Willersinn, MD, Alpha-1 Plus, Belgium Marion Bouchecareilh, University of Bordeaux, Alpha-1 Liver Research, France Robert Durlik, Alpha-1 Poland, Founder, Poland Robert Kroesen, Patient Representative, Panama Tim Frost, AlphaNet Board Member, USA

www.alpha-1global.org

Presenting Sponsors: AlphaNet | CSL Behring | Grifols Gold Sponsors: Arrowhead | Kamada | Vertex

June 2019 Jun. 1 Dakotaland Alphas Jun. 4 Northern Indiana Alphas Jun. 6 Sacramento Alphas Jun. 8 Midwest Alphas

Northwest Indiana Alphas

Glass City, Toledo Alphas Jun. 13 Alpha Pack Support Group Jun. 18 Oklahoma Alphas

Arkansas Alphas Jun. 29 Illiana Alphas July 2019 Jul. 13 Chicagoland Alphas

Virginia Alpha-1 Connection

Alabama Alphas

Alpha Opportunities

Casper Alphas

Open Arms for Alpha-1 SG Jul. 20 Oregon Alphas August 2019 Aug. 3 Alpha-1 Atlanta

Heart of Georgia Alphas

Dakotaland Alphas Aug. 4 Denver Alpha Beaters Aug. 10 Southeast Keystone Alphas

Norfolk, VA Alpha-1 Connection

Glass City, Toledo Alphas

FL Gulf Coast Alphas

Northwest Indiana Alphas Aug. 15 Alpha Pack Support Group Aug. 16 Arkansas Alphas Aug. 17 North Alabama Alphas

Alphas of Northern Illinois Aug. 24 Illiana Alphas

Sacramento Alphas Aug. 31 Bayou City Alphas

Sioux Falls, SD Ft. Wayne, IN Roseville, CA Kansas City, MO Dyer, IN Toledo, OH Milwaukee, WI Oklahoma City, OK Benton, AR Terre Haute, IN Wood Dale, IL Richmond, VA Birmingham, AL Paoli, PA Douglas, WY Peoria, IL Portland, OR Johns Creek, GA Macon, GA Sioux Falls, SD Littleton, CO Boyertown, PA Norfolk, VA Toledo, OH Sarasota, FL Fort Wayne, IN Milwaukee, WI Benton, AR Huntsville, AL Rockford, IL Terre Haute, IN Rocklin, CA Houston, TX

2019 CALENDAR

SUPPORT GROUP MEETINGS April 2019 Apr. 3 San Diego Alphas Del Mar, CA Apr. 4 Chattanooga Area Alphas Chattanooga, TN Apr. 6 Dallas/Fort Worth Alphas Kennedale, TX

Southeast Louisiana Alphas Covington, LA

Gainesville, FL Alphas Dunnellon, FL

SoCal Roadrunners Palm Desert, CA

Hudson Valley, NY Alphas Middletown, NY

West Virginia Alphas Princeton, WV Apr. 10 Alphazonies Scottsdale, AZ Apr. 11 Massachusetts Alphas Needham, MA

Alpha Pack Support Group Milwaukee, WI Apr. 13 Norfolk, VA Alpha-1 Connection Norfolk, VA

FL Gulf Coast Alphas Sarasota, FL

Buckeye Alphas Columbus, OH

Northwest Indiana Dyer, IN

Dakotaland Alphas Sioux Falls, SD

Alpha-1 Naples Naples, FL

Greater St. Louis Area Alphas St. Louis, MO Apr. 18 Alpha-1 Mainers Lewiston, ME Apr. 20 Alphas of Northern Illinois Rockford, IL

Tampa FL Alphas Lutz, FL

Alpha-1 Atlanta Alpharetta, GA Apr. 23 Oklahoma Alphas Oklahoma City, OK Apr. 27 Heart of Georgia Alphas Macon, GA

Bayou City Alphas Houston, TX May 2019 May 4 Alabama Alphas Birmingham, AL

Maryland Alphas Ownings Mill, MD

Coastal Carolina Alphas Charleston, SC

AlphaBurgherZ Monroeville, PA

Dallas/Fort Worth Alphas Kennedale, TX May 11 Alpha Gators Weston, FL

Chicagoland Alphas Wood Dale, IL

Virginia Alpha-1 Connection Richmond, VA

NY/NJ Alphas Fort Lee, NJ

Open Arms for Alpha-1 SG Peoria, IL

San Antonio/Austin Alphas San Marcos, TX May 14 Southwest Missouri Alphas Joplin, MO May 18 North Alabama Alphas Huntsville, AL

Reno/Tahoe Alphas Reno, NV

Capital Region of NY Alphas Albany, NY

Southern Colorado Alphas Pueblo, CO

Kentucky Alphas Shepherdsville, KY May 20 Idaho Community Outreach SG Meridian, ID May 21 Big Red Alphas Papillion, NE May 23 Chattanooga Area Alphas Chattanooga, TN May 30 Alpha-1 Mainers Boston, MA

Oklahoma Alphas Oklahoma City, OK

For more information about Support Group Meetings, contact Barbee Bennington, (877) 228-7321, ext. 227 or bbennington@alpha1.org.

ALPHA-1 EDUCATION DAYS May 5 Portland, OR Aug. 3 Denver, CO Sept. 28 Minneapolis, MN Oct. 26 Philadelphia, PA For more information about Education Days, contact Kim Caraballo, (877) 228-7321, ext. 323 or ycaraballo@alpha1.org.

BUILDING FRIENDS FOR A CURE (BFC) EVENTS Apr. 4 Jonathan Maidment’s Hiking for a Cure Pacific Coast, USA Angela McBride amcbride@alpha1.org May 4 Step Forward for Alpha-1 Portland Portland, OR Angela McBride amcbride@alpha1.org May 4 Hero Walk Henrico, VA Pam Vanscoy vaalpha1herowalk@yahoo.com May 11 George Washington Bridge Walk New York/New Jersey Joe Reidy JoeReidy@Verizon.net Jun. 8 Iowa Scoop/Walk Johnston, IA Peg Iverson pegiver@mchsi.com Aug. 3 Step Forward for Alpha-1 Colorado - Denver, CO Angela McBride amcbride@alpha1.org Aug. 10 Alpha-1 Duck Race Boyertown, PA Larry Hoffman jlremodel@hotmail.com Aug. 16 Drive for Awareness Willingboro, NJ Kelly Aaronson Kellyaaronson@yahoo.com Aug. 17 Alpha-1 Duck Race Reading, PA Larry Hoffman jlremodel@hotmail.com Sept. 20-22 Escape to the Cape Cape Cod, MA Angela McBride amcbride@alpha1.org Nov. Alpha-1 Awareness Month Virtual Walks USA Angela McBride amcbride@alpha1.org For more information on Building Friends for a Cure, please contact Angela McBride, (877) 228-7321 ext. 233 or amcbride@alpha1.org.

SPECIAL EVENTS Apr. 29 Celebration of Life Golf Classic and Dinner Reception Miami, FL Linda Rodriguez lrodriguez@alpha1.org May 20 Alpha-1 Awards Reception at ATS Dallas, TX Angela McBride amcbride@alpha1.org Jun. 6 Alpha-1: A Model for Philanthropy New York, NY Angela McBride amcbride@alpha1.org For more information on Special Events, please contact Linda Rodriguez, (877) 228-7321 ext. 237 or lrodriguez@alpha1.org

Please visit the calendar at alpha1.org for more information on these support group meetings and other upcoming events. www.alpha1.org

27

ALPHA-1 1 FOUNDATION

NONPROFIT ORG U.S. POSTAGE PAID MIAMI, FL PERMIT # 8124

3300 Ponce de Leon Blvd. • Coral Gables, FL 33134

Alpha-1 research studies enrolling n Alpha-1 Carbamazepine Study Study for ZZ or SZ Alphas between 14-80 years old with severe liver disease to test if the drug carbamazepine improves liver disease.

Visit https://clinicaltrials.gov/ct2/show/NCT01379469 for more information. n Alpha-1 Foundation Liver Study Study for Adult ZZ Alphas to determine that causes and progression of liver disease. “Known Severe Liver Disease” and “Post-Transplant” groups still enrolling. (Liver Biopsy groups ended enrollment).

Visit https://clinicaltrials.gov/ct2/show/NCT02014415 for more information. n Alvelestat for the Treatment of Alpha-1 The National Institutes of Health (NIH)/National Center for Advancing Translational Science (NCATS) and Mereo Biopharma are sponsoring an ongoing study that seeks to test the safety and efficacy of the pill Alvelestat, and investigational drug treatment.

This study will test how Alvelestat affects lung damage related to alpha-1 antitrypsin deficiency. Eligible participants must be 25-80 years of age with a genotype of SZ, ZZ, SNull, or ZNull. Participants receiving augmentation therapy must be willing to stop for at least 12 weeks before treatment. Those eligible to participate will receive either Alvelestat or placebo for 12 weeks. Multiple centers in the U.S. are recruiting. Please visit https://clinicaltrials.gov/ct2/show/ NCT03679598 for more information. n COPD Exacerbation Study Columbia University (NY) study for adults aged 35-80 with alpha-1 deficiency or alpha-1 carrier state AND physician diagnosed COPD Visit https://recruit.cumc.columbia.edu/clinical_trial/1523

n GLASSIA Study Augmentation therapy study for severely deficient Alphas. Multiple sites recruiting. The study duration is six months. Visit https:// clinicaltrials.gov/ct2/show/NCT02525861 for more information. n Hyaluronic Acid Inhalation Study Inhaled therapy study for severely deficient Alphas diagnosed with emphysema. Multiple sites recruiting. Visit https://clinicaltrials.gov/ ct2/show/NCT03114020 for more information. n Hyperpolarized Xenon Gas MRI for Assessing Pulmonary Function Study Duke University (NC) study for adults 18-80 with Alpha-1 genotype of MZ who have a smoking history of less than 5 pack-years and have normal lung function. Visit: https://clinicaltrials.gov/ct2/show/ NCT01280994 for more information. n Lung Imaging Study Columbia University (NY) study for adults aged 45-80 with Alpha-1 or Alpha-1 carrier state with OR without COPD. Visit https:// clinicaltrials.gov/ct2/show/NCT02978144 for more information. n O2VERLAP Study COPD Foundation and American Sleep Apnea Association study for adults aged 40 and up diagnosed with both COPD and Obstructive Sleep Apnea (OSA), and with a prescription for CPAP therapy. Study is testing a “virtual classroom” where patients can learn how to best use their machines and improve their self-care. Visit https://clinicaltrials.gov/ct2/ show/NCT03446768 or www.O2VERLAP.org for more information. n SPARTA Study Augmentation therapy study for severely deficient Alphas. Multiple sites recruiting. The study duration is 3 years.

Visit https://clinicaltrials.gov/ct2/show/study/NCT01983241 for more information.