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Novel Therapeutic Uses of Alpha-1 Antitrypsin: A Window to the Future.
MedLine Citation:
PMID:  23205701     Owner:  NLM     Status:  Publisher    
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.
Adam Wanner; Adriana De Arce; Erin Pardee
Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2012-12-4
Journal Detail:
Title:  COPD     Volume:  -     ISSN:  1541-2563     ISO Abbreviation:  COPD     Publication Date:  2012 Dec 
Date Detail:
Created Date:  2012-12-4     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101211769     Medline TA:  COPD     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
1Alpha-1 Foundation , Miami, Florida , USA.
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