Dr. Igor Barjaktarevic on Advancing Alpha-1 Care

Advancing Alpha-1 Antitrypsin Deficiency Care: Insights from Pulmonologist Dr. Igor Barjaktarevic

Alpha-1 antitrypsin deficiency (Alpha-1 or AATD) remains widely underdiagnosed despite its significant impact on lung and liver health. Dr. Igor Barjaktarevic, a pulmonologist and researcher at UCLA Health, has been instrumental in shifting the perception of AATD from a late-stage COPD diagnosis to a condition requiring early detection, proactive treatment and individualized patient care. His research has helped expand the medical community’s approach to Alpha-1 management, with ongoing efforts to improve screening and treatment options.

As the principal investigator for UCLA’s Alpha-1 biomarker study, Dr. Barjaktarevic is at the forefront of research aimed at identifying new biomarkers to enhance early detection and therapeutic interventions. His work, including the co-authored review Management of Lung Disease in Alpha-1 Antitrypsin Deficiency: What We Do and What We Do Not Know, highlights evolving approaches to patient care and disease progression.

With increased attention on emerging therapies and patient advocacy, events like the upcoming Alpha-1 Foundation National Conference in June provide a platform for discussions on improving diagnosis, expanding access to treatment and advancing research efforts.

Early diagnosis and the evolution of AATD management

For years, AATD was primarily associated with advanced chronic obstructive pulmonary disease (COPD), and the focus was often on survival rather than understanding disease progression and quality of life improvements. Dr. Barjaktarevic and his colleagues have helped reframe this approach by emphasizing the importance of screening, early intervention, personalized treatment strategies and lung function preservation.

“We started by recognizing Alpha-1 as advanced COPD that progresses rapidly. But over time, we have moved beyond to a deeper understanding — that Alpha-1 antitrypsin deficiency is not a disease but a predisposition to multiple diseases and that early diagnosis can allow us to intervene in timely fashion and minimize or even prevent the disease development.” Dr. Barjaktarevic explains.

Despite guidelines recommending AATD testing in patients with COPD and unexplained liver disease, many cases remain undiagnosed. Expanding screening efforts — particularly among primary care providers and pulmonologists — is still a critical step in ensuring intervention for patients who could receive help from treatment.

Emerging therapies and the future of Alpha-1 treatment

Dr. Barjaktarevic 's research focuses on biomarkers, gene therapies and novel treatment approaches that aim to modify the disease trajectory rather than simply manage symptoms.

“We are now exploring therapies that go beyond just slowing an already advanced lung disease progression — we are tackling AATD from different angles with very different mechanism of action of investigated therapies. Ongoing clinical trials investigate the approaches aiming to protect lung, liver, or both systems together at the same time; various methods of systemic or intrapulmonary augmentation of AAT, with human-derived or recombinant proteins; advanced gene-therapy approaches targeting DNA or RNA which are being developed may, if proven successful, both treat the diseased ones but also may represent an early risk-modifying strategies for many individuals with AAT in earlier life,” he notes.

Current treatments, such as augmentation therapy, help maintain protective levels of Alpha-1 antitrypsin in the lungs, but they do not reverse existing damage. The next frontier in AATD treatment includes:

• Recombinant therapies designed to lengthen dosing frequency.
• Inhalable and subcutaneous formulations for more convenient therapy delivery.
• Therapies aiming to establish better balance between proteases and anti-proteases.
• Gene-based therapies that could treat lung and/or liver disease or prevent them from developing at all.

With clinical research expanding in these areas, there is renewed optimism about more effective and accessible treatments becoming available in the coming years.

The role of patient advocacy and the Alpha-1 Foundation National Conference

Beyond clinical advancements, patient advocacy has played a crucial role in shaping the future of AATD care. Organizations like the Alpha-1 Foundation have helped drive awareness, policy changes and research funding.

“The Alpha-1 Foundation is a model for how patient-driven advocacy can change the landscape of disease research and treatment by connecting scientists, clinicians, regulators and industry in one solid network,” Dr. Barjaktarevic states.

The annual Alpha-1 Foundation National Conference serves as an important gathering for patients, prescribers, researchers and other AATD community members to discuss advancements in diagnosis, treatment and disease management strategies. Events like this help bridge the gap between clinical research, real-world patient experiences and specialty care.

The future of AATD care and the importance of collaboration

As research progresses, collaboration across the health care ecosystem remains vital. Physicians, researchers, advocacy groups and specialty care providers all play a role in ensuring that patients receive timely diagnoses and have access to evolving treatment options.

Dr. Barjaktarevic's work continues to advance the field of AATD, helping shift the conversation toward earlier detection and innovative therapies. As new treatment options emerge, ensuring that patients, caregivers and health care providers stay informed and engaged will be critical to improving outcomes.

With continued advancements in research, treatment access and patient advocacy, the future of AATD care is evolving toward earlier diagnosis, better management and disease prevention.

Comprehensive support for Alpha-1 patients

For patients managing Alpha-1, access to the right therapy and ongoing support is critical. Accredo has access to all Alpha-1 specialty therapies available on the market, ensuring that patients receive the option best suited to their needs.

Through our specialized Therapeutic Resource Center® (TRC) model, Accredo provides dedicated support from specialty-trained pharmacists and nurses who understand the complexities of AATD treatment. Whether managing medication adherence, navigating insurance or addressing patients with social determinants of health (SDoH) concerns, the TRC model helps ensure that every patient receives the care and support they need to optimize their treatment journey.

For more information on Alpha-1 treatment options and specialized patient support, visit Accredo.com.