About Us
The Department of Genetic Medicine at Weill Cornell leads a dynamic and innovative translational research program, advancing diverse fields such as Genetic Therapy and Personalized Medicine.
Evaluation of tamoxifen as a therapy to augment alpha-1-antitrypsin concentrations in Z homozygous alpha-1-antitrypsin-deficient subjects.
| Publication Type | Academic Article |
| Authors | Wewers M, Brantly M, Casolaro M, Crystal R |
| Journal | Am Rev Respir Dis |
| Volume | 135 |
| Issue | 2 |
| Pagination | 401-2 |
| Date Published | 02/01/1987 |
| ISSN | 0003-0805 |
| Keywords | Homozygote, Tamoxifen, alpha 1-Antitrypsin Deficiency |
| Abstract | Tamoxifen, an agent that binds to intracytoplasmic estrogen receptors, was evaluated as a possible means of increasing alpha-1-antitrypsin (alpha 1AT) synthesis and/or secretion and thus alpha 1AT serum levels in subjects with the homozygous form of alpha 1AT deficiency. Administration of tamoxifen (10 mg twice daily) to 30 Z homozygotes for a 30-day period was not associated with adverse reactions. However, although serum alpha 1AT levels increased significantly (p less than 0.03), the increase was minor (average pretreatment levels, 32 +/- 1 mg/dl; levels at 30 days of therapy, 35 +/- 1 mg/dl) and far below the "threshold" level of 80 mg/dl considered "protective" against an increased risk for emphysema. Thus, while the concept that increasing alpha 1AT synthesis and/or secretion is a rational goal for treating the Z homozygous form of alpha 1AT deficiency, tamoxifen will not be useful in this regard. |
| DOI | 10.1164/arrd.1987.135.2.401 |
| PubMed ID | 3492949 |