Standard of Care News & Updates

Breaking News: Kamada Ltd. announced the discontinuation of its Phase 3 InnovAATe clinical trial studying inhaled Alpha-1 Antitrypsin therapy.

What This Means:

While disappointing, this development highlights the ongoing challenges in developing alternative AAT delivery methods. The Alpha-1 Foundation thanked the patients who participated in this important clinical trial, emphasizing that their dedication advances the field even when trials don't achieve their primary endpoints.

Our Take: This underscores the continued need for research into optimal AAT formulations and delivery methods. The Mark Egly Foundation supports diverse approaches to making AAT therapy more effective and accessible.

🔗 Read the Alpha-1 Foundation announcement

Expanding Access: The University of Kentucky has been officially designated as a Clinical Resource Center (CRC) by the Alpha-1 Foundation, expanding the network of specialized AATD care facilities.

Significance: Clinical Resource Centers provide comprehensive, multidisciplinary care for individuals with AATD, bringing together pulmonologists, hepatologists, genetic counselors, and other specialists. This designation improves access to expert care for patients in Kentucky and surrounding regions.

Impact: More CRCs mean better access to diagnosis, treatment, and specialized care—exactly what the Mark Egly Foundation advocates for in changing standards of care.

🔗 Learn more about the UK CRC designation

Global Collaboration: The second Latin American Alfa-1 Meeting, titled "Consolidating Advances, Expanding Horizons," was held in Buenos Aires, Argentina, bringing together clinicians, researchers, and patient advocates from across Latin America.

Why It Matters: AATD is a global issue requiring international collaboration. This meeting demonstrates growing awareness and improved infrastructure for AATD diagnosis and treatment in Latin America—regions that have historically had limited access to specialized care.

🔗 Read about the Argentina meeting

Empowering Patient Voices: The Alpha-1 Foundation launched the Fred C. Walsh Speaker Bureau to amplify patient voices and elevate patient-centered work in research, policy, and healthcare delivery.

Connection to Our Mission: Patient advocacy is central to changing standards of care. When patients share their stories with physicians, policymakers, and the public, they drive awareness and systemic change. The Mark Egly Foundation strongly supports patient-led advocacy efforts.

🔗 Discover the Speaker Bureau

According to ClinicalTrials.gov, there are currently 289 clinical studies related to Alpha-1 Antitrypsin registered globally. These studies span:

Gene Therapy

• Multiple AAV-based gene therapy trials aiming to enable patients to produce their own functional AAT
• CRISPR-based approaches in preclinical development
• Long-term safety and efficacy studies

Novel Augmentation Therapies

• New formulations with improved pharmacokinetics
• Alternative delivery methods (inhaled, subcutaneous)
• Dose optimization studies

Expanded Indications

• AAT in autoimmune diseases
• AAT for organ transplant rejection prevention
• AAT in acute inflammatory conditions
• AAT in neurological disorders

Biomarker Development

• Improved diagnostic tools
• Prognostic markers for disease progression
• Monitoring tools for treatment response

🔗 Search Alpha-1 Antitrypsin clinical trials

The Alpha-1 Foundation continues to expand its global leadership role, recently highlighting its strategic engagement with the European Union to improve AATD awareness, diagnosis, and treatment across Europe.

Policy Priorities:

• Expanded newborn screening programs
• Insurance coverage for augmentation therapy
• Funding for AATD research
• Clinical guideline updates
• Healthcare provider education initiatives

What We're Watching:

• FDA approval pathways for new AAT therapies
• Insurance coverage expansions
• Professional society guideline updates
• International regulatory harmonization

🔗 Read about EU strategic engagement

The Mark Egly Foundation is developing a comprehensive program to transform how patients and families navigate Alpha-1 Antitrypsin Deficiency:

Early Identification

• Promoting routine screening for at-risk populations
• Educating physicians on when to test for AATD
• Supporting accessible, affordable testing options
• Reducing time to diagnosis from years to months

Genetic Counseling

• Understanding test results and implications
• Family cascade testing guidance
• Carrier state counseling
• Reproductive planning support

Treatment Navigation

• Understanding augmentation therapy options
• Insurance authorization assistance
• Finding specialized healthcare providers
• Accessing Clinical Resource Centers

Comprehensive Care Coordination

• Connecting pulmonary, hepatic, and other specialty care
• Monitoring disease progression
• Managing comorbidities
• Preventive care strategies

Clinical Trial Matching

• Identifying relevant research opportunities
• Understanding trial participation
• Connecting patients with researchers
• Supporting informed decision-making

Patient Education Resources

• Understanding AATD and its systemic effects
• Lifestyle modifications and environmental protections
• Recognizing symptoms requiring attention
• Staying current with research and treatments

Family Education

• Risk assessment for family members
• When and how to get tested
• Supporting affected loved ones
• Inheritance patterns and genetic implications

Healthcare Provider Education

• Our "Uniting Doctors" initiative
• CME programs and conferences
• Case studies and clinical pearls
• Latest research and treatment guidelines

Peer Support Networks

• Connecting patients with similar experiences
• Online and in-person support groups
• Mentorship from experienced "Alphas"
• Family and caregiver support

Advocacy Training

• Becoming an effective self-advocate
• Sharing your story to drive change
• Engaging with policymakers
• Participating in awareness campaigns

Events and Engagement

• Educational conferences and webinars
• Awareness walks and fundraising events
• Research symposia
• Patient registry participation

Alpha-1 Research Registry

• Contributing to research without leaving home
• Accelerating scientific discoveries
• Being contacted for relevant clinical trials
• Advancing knowledge that helps everyone

Clinical Trial Opportunities

• Access to cutting-edge therapies
• Contributing to future treatments
• Comprehensive monitoring and care
• Potential therapeutic benefit

The Alpha-1 Foundation provides monthly updates on peer-reviewed research publications funded by their research program. Recent publications cover:

• Genetic variants and clinical correlations
• Biomarker discovery and validation
• Treatment outcomes and safety data
• Novel therapeutic approaches
• Quality of life studies

🔗 View November PubMed articles

1. Physician Education Campaign

• Targeting 10,000+ physicians by 2030
• CME-accredited programs
• Case-based learning modules
• Practice-ready diagnostic algorithms

1. Medical School Curriculum Integration

• Working with every medical schools
• Incorporating AATD into standard teaching
• Training the next generation of physicians
• Earlier identification, better outcomes

1. Insurance Coverage Advocacy

• Fighting arbitrary treatment restrictions
• Expanding testing coverage
• Advocating for preventive therapy coverage
• Ensuring equitable access

1. Clinical Guideline Evolution

• Partnering with professional medical societies
• Advocating for expanded diagnostic criteria
• Promoting earlier treatment initiation
• Recognizing systemic manifestations

1. Alternative Production Research

• Funding plant-based AAT development
• Supporting gene therapy advancement
• Enabling cost reduction and scalability
• Making therapy accessible globally

November 2025 marked another successful Alpha-1 Awareness Month, with the Alpha-1 Foundation celebrating its 30th anniversary. The community came together to:

• Share personal stories and raise awareness
• Participate in walks and fundraising events
• Educate healthcare providers and the public
• Honor those lost to AATD and celebrate survivors

Impact: Awareness campaigns are crucial for earlier diagnosis. Every person who learns about AATD becomes a potential advocate who might recognize symptoms in themselves or others.

🔗 Read Week 4 and Week 5 recaps

The Alpha-1 community mourns the loss of Dr. Gordon Cadwgan, PhD, who passed away peacefully on November 29, 2025, at 81 years old. Dr. Cadwgan was a dedicated researcher and advocate whose work advanced the understanding and treatment of AATD.

His legacy reminds us why this work matters and why we must continue the fight until everyone affected by AATD has access to diagnosis, treatment, and hope.

🔗 Remember Dr. Cadwgan

January 2026: Virtual Education Day & State of the Union

The Alpha-1 Foundation will host its 2026 Virtual Education Day and State of the Union on January 28, 2026, providing updates on research, advocacy, and community initiatives.

🔗 Register for the 2026 Virtual Event

Upcoming Research Milestones

• Gene therapy trial results expected
• New augmentation therapy formulations in development
• Expanded clinical trials for autoimmune applications
• Alternative production method advancements

Policy Developments

• Updated clinical practice guidelines anticipated
• Insurance coverage expansion efforts ongoing
• International regulatory harmonization progress
• Newborn screening program expansions

For Patients:

• Share your story
• Join the Alpha-1 Research Registry
• Participate in advocacy
• Connect with community resources

For Healthcare Providers:

• Test more liberally for AATD
• Join our "Uniting Doctors" network
• Stay current with latest research
• Refer patients to specialized centers

For Everyone:

• Spread awareness
• Support research funding
• Advocate for policy changes
• Build community connections

Mark Egly Foundation

• Subscribe to our newsletter
• Follow us on social media
• Attend our educational events
• Join our research initiatives

Alpha-1 Foundation Resources

• Monthly research updates
• Clinical trial notifications
• Policy and advocacy alerts
• Community event calendar

🔗 Alpha-1 Foundation News

The standard of care is evolving. Stay informed, stay engaged, and be part of the transformation.

Together, we're building a future where Alpha-1 Antitrypsin Deficiency is diagnosed early, treated effectively, and understood comprehensively—changing lives and saving families from preventable suffering.