Gene therapy looks promising for alpha-1-antitrypsin deficiency

Reported by Susan Aldridge, PhD, medical journalist

Clinical trial shows that gene therapy for alpha-1-antitrypsin deficiency is safe and may also produce the desired therapeutic protein.
Alpha-1-antitrypsin (AAT) is a protein made in the liver which protects the lungs by blocking inflammatory products which can destroy lung tissue. Around 100,000 Americans inherit a deficiency in AAT which leaves them with extensive lung damage. Often, AAT deficiency goes undiagnosed. However, it is potentially treatable with gene therapy - where a copy of the normal gene for AAT would be delivered into the body to make up for the deficiency.

Researchers at the University of Florida now report on a clinical trial involving 12 patients with AAT deficiency. They delivered a so-called gene vector, a virus which carries the AAT gene into the body. This was shown to be safe and a higher dose of the virus was able to introduce the gene into the bloodstream. In one patient, there was also evidence that AAT protein was actually produced, although not at high enough a level to be therapeutic.

The next step would be to proceed to a vector that may be more efficient at making the protein. If trials show therapeutic levels of AAT can be achieved through gene therapy, it would be an important breakthrough for these patients. Currently, they must have weekly injections of AAT derived from human plasma. The treatment, which has to be lifelong, does not cure the disease, but may slow its progression.

Source
Human Gene Therapy online 21st November 2006

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