Alpha(1)-Antitrypsin Therapy

Currently, there are a number of approved therapies for the treatment of panacinar emphysema, a disease that results from severe deficiency of circulating alpha(1)-antitrypsin (AAT). Each of these products is manufactured by purification from human plasma, with the standard mode of delivery being weekly infusion of the long-lived protein. The adoption of AAT therapy in the treatment of hereditary emphysema and the well-known activity of AAT as an inhibitor of human neutrophil elastase (HNE) has suggested its use in other indications where unregulated HNE may be a causative factor. These include cystic fibrosis, chronic lung disease of prematurity and other, more common forms of chronic obstructive pulmonary disease, such as smoking-related emphysema. Other inhibitory pathways that are known for AAT have suggested its further potential in asthma therapy. A growing understanding of global mechanisms involved in respiratory disease progression, together with advances in the inhalation delivery of AAT, suggest that AAT therapy may expand beyond its current use as an infusion therapy for AAT deficiency.