title: Novel therapeutic uses of alpha-1 antitrypsin: a window to the future.
abstract: Alpha-1 antitrypsin, a potent serine protease inhibitor, has been used as augmentation therapy in patients with alpha-1 antitrypsin deficiency for many years. Recent research into the diverse anti-inflammatory, immune-modulatory and tissue-protective actions of alpha-1 antitrypsin has raised the possibility of broadening the therapeutic spectrum of alpha-1 antitrypsin to include diseases other than alpha-1 antitrypsin deficiency. The purpose of the workshop was to summarize the results of basic investigations and, if available, clinical studies in which the effects of alpha-1 antitrypsin were explored in relation to clinical conditions that are not associated with alpha-1 antitrypsin deficiency. Included among these are type 1 diabetes, cell/organ rejection, viral infection, cystic fibrosis, bronchiectasis/COPD, heart failure, Crohn's disease and connective tissue diseases. Although the therapeutic utility of alpha-1 antitrypsin in these conditions remains to be established, the existing data suggest that this protein eventually will become a treatment option in several diseases some of which are not rare. At present, only human plasma-derived alpha-1 antitrypsin is available for clinical use. Given the limited supply and the potential for extended use of this product, there will be a need for new formulations of alpha-1 antitrypsin in the future. Therefore, the prospect of finding new sources and airway delivery methods of alpha-1 antitrypsin were also discussed. The presentations at the meeting addressed the scientific basis for new clinical applications of alpha-1 antitrypsin and the regulatory requirements needed to bring this therapeutic protein to a wider range of patient populations.
authors.affiliation: Alpha-1 Foundation, Miami, FL 33133, USA. awanner@alpha-1foundation.org
authors.forename: Adam
authors.initials: A
authors.lastname: Wanner
authors.forename: Adriana De
authors.initials: AD
authors.lastname: Arce
authors.forename: Erin
authors.initials: E
authors.lastname: Pardee
mesh_terms: D000818:Animals; D000894:Anti-Inflammatory Agents, Non-Steroidal / Q000627:therapeutic use*; D003922:Diabetes Mellitus, Type 1 / Q000188:drug therapy*; D017277:Drug Approval; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D008171:Lung Diseases / Q000188:drug therapy*; D015202:Protein Engineering; D011994:Recombinant Proteins / Q000627:therapeutic use; D015842:Serine Proteinase Inhibitors / Q000627:therapeutic use*; D014481:United States; D014777:Virus Diseases / Q000188:drug therapy*; D000515:alpha 1-Antitrypsin / Q000627:therapeutic use*
chemical_list: D000894:Anti-Inflammatory Agents, Non-Steroidal; D007155:Immunologic Factors; D011994:Recombinant Proteins; D015842:Serine Proteinase Inhibitors; D000515:alpha 1-Antitrypsin