Alpha-1 Antitrypsin as a Therapeutic Agent for Conditions not Associated with Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin is a positive acute phase reactant whose serum level rises in response to inflammatory stress, presumably to balance pro-inflammatory processes. In addition to its serine protease inhibitory action, alpha-1 antitrypsin exhibits broader anti-inflammatory and immunomodulatory activity, and increasing its serum concentration by the administration of exogenous alpha-1 antitrypsin to above-normal levels potentially could be therapeutic in conditions other than alpha-1 antitrypsin deficiency. In vitro observations, studies in animal models and in some instances early human trials suggest that intravenous or inhaled alpha-1 antitrypsin has beneficial effects in type 1 diabetes, viral infections, graft-versus-host disease, cystic fibrosis, and alpha-1 antitrypsin-replete chronic obstructive pulmonary disease among others. While the results of pivotal clinical trials have not been reported to date, new indications for alpha-1 antitrypsin therapy are likely to emerge in the future based on currently available scientific data.