Although patients showed some elevated immune response to the gene therapy vector which is designed to quickly break down after delivering its cargo researchers did not detect any evidence that the patients' bodies rejected the transferred genes or the newly created protein.

"That's a really good sign," said Brantly, a member of the Powell Gene Therapy Center and the UF Genetics Institute, who sees about 150 alpha-1 patients in his medical practice. "After we gave the injections, the individuals stayed on the ward for five days while we monitored them. There were no ill effects, only a minimal amount of redness, and by the end of the five days most of the subjects were actually bored."

Currently, the only limitedly effective treatment for patients with serious breathing symptoms involves weekly intravenous injections of alpha-1 protein derived from human plasma. The injections must continue throughout a patient's life, according to the American Lung Association. They do not cure the disease, but they do appear to slow its progression.

"This study gives us encouraging evidence that gene therapy for alpha-1 is a realistic possibility," said John Walsh, president and chief executive officer of the nonprofit Alpha-1 Foundation, which has been supporting research of this kind for more than a decade. "The augmentation therapy available now has slowed down the progression of our lung disease and extended many of our lives. The hope of gene therapy is that we may have a one-time, brief series of injections that could allow our own bodies to produce the alpha-1 protein we need to live a normal lifetime. The alpha-1 community is incredibly grateful for the progress that these dedicated investigators have made," Walsh said.

www.umassmed