A clinical trial at the University of Florida has evaluated a gene therapy to treat Alpha-1 antitrypsin deficiency, and the results are promising. People with Alpha-1 antitrypsin deficiency make little or none of the alpha-1 antitrypsin protein, which is essential for health. In this clinical trial, the researchers used a virus to deliver a corrective gene that codes for the alpha-1 antitrypsin protein to 12 participants with Alpha-1 antitrypsin deficiency.
“The primary end point in the trial was to see whether it was safe to give patients this gene transfer vector and then to try to begin to see if we could get the dose into a range where we would begin to replace the missing protein in the blood,” said Dr. Terence Flotte, a pediatrician, geneticist and microbiologist with UF’s College of Medicine and a member of the Powell Gene Therapy Center and the UF Genetics Institute. “We found that we can use this agent safely and we also saw evidence in the patients’ blood that the higher doses successfully introduced the vector DNA. In one patient we saw evidence for a very brief period that some of the alpha-1 protein was being produced, but not at a high enough level to be beneficial.” (from the press release from University of Florida)