An international conference on Therapy for Alpha 1 -Antitrypsin Deficiency is being organized by the Alpha One Foundation on June 15-18 1999 at Haverford College in Haverford Pennsylvania. The goal of this conference is to stimulate the research and development of new therapies for the treatment of lung disease resulting from alpha 1 -antitrypsin deficiency (AATD). AATD is a rare disorder with an estimated gene frequency in Americans of Northern European origin comparable to that of cystic fibrosis occurring in the range of 1 in 1500 to 1 in 2750 live births. AATD, an inherited, recessive, genetic disease, is an excellent model for destructive lung disease, for which there is a well- established body of scientific information. Careful evaluation of the biological basis of lung inflammation and destruction is the cornerstones for the development of novel therapies for AATD. Topics related to the biology of lung destruction will include markers of lung destruction, markers of lung inflammation and strategies for clinical studies. Markers are critical to the development of novel therapies since they will permit studies of """"""""process"""""""" as well as """"""""outcome"""""""" of therapy on the development of destructive lung disease. Potential therapies to be reviewed in depth will include IV and aerosol alpha1AT augmentation, retinoic acid treatment, modulation of protein transport, the development of synthetic inhibitors of inflammation and gene therapy. A majority of the proposed conference participants are doing cutting edge research, which is highly relevant to the treatment of A1AD, including international investigators working in the basic sciences, clinical sciences in industry. Representatives of the United States Food and Drug Administration and the National Institutes of Health will participate as well. Some members of the Alpha One Foundation with AATD who suffer from COPD will be present as observers and to provide patient input to the ongoing and proposed work of the investigators. The major issue this conference addresses is the fact that there have been no new therapies for the treatment of AATD in the last decade. This is notwithstanding the fact that significant advances have occurred in the scientific understanding of the relationship among the research areas on lung destruction, lung inflammation and the biology of AATD. The conference will provide a unique and timely forum that brings together researchers, industry and government leaders to exchange and disseminate new information and will foster new collaborations for the development of more effective drug therapies for this genetic disorder.
