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Alpha 1-antitrypsin deficiency is an inherited disorder characterized by low blood and lung levels of alpha 1-antitrypsin, a protein and carbohydrate compound that inhibits proteolytic or protein-degrading enzymes. Alpha 1-antitrypsin protects against neutrophil elastase, a proteolytic enzyme capable of destroying the walls of the alveoli or air sacs of the lungs. A deficiency in alpha 1-antitrypsin results in the loss of anti-neutrophil-elastase protection of the lower respiratory tract and leads to the development of emphysema, the distention of the lungs by gas or air. The effectiveness of an aerosol preparation of purified human plasma alpha 1-antitrypsin in improving anti-neutrophil elastase defenses in the lower respiratory tract was assessed in 12 patients with a deficiency of alpha 1-antitrypsin. The aerosol preparation increased alpha 1-antitrypsin levels in the epithelial cells lining the lung from 0.28 micromole (uM) before therapy to 5.86 uM after therapy. Anti-neutrophil-elastase capacity was increased from 0.78 uM before therapy to 4.16 uM after therapy. Aerosolized alpha 1-antitrypsin was shown to diffuse from the lung epithelial cells to cells within the lung tissue and eventually to the bloodstream, and was not associated with any side effects. Thus, short-term use of aerosolized alpha 1-antitrypsin is both safe and effective in treating alpha 1-antitrypsin deficiency, and improves anti-neutrophil-elastase defenses in the lower respiratory tract. (Consumer Summary produced by Reliance Medical Information, Inc.)