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Current Treatments

AAT replacement therapy—also called augmentation therapy—was first used in 1987 to raise the level of AAT in the blood, thus ensuring adequate levels in the lungs to neutralize the enzyme neutrophil elastase known to cause most of the damage. The treatment has been developed using human plasma that has gone through a rigorous process to ensure no viruses can be transmitted along with the AAT. The therapy is administered via an IV infusion once every week or two, and it must continue throughout a person’s lifetime in order to be effective. While the treatment cannot cure the disease, it does appear to slow its progression. The three current augmentation therapy products available are Prolastin™, Aralast™, and Zemaira™.

In addition to augmentation therapy, several new treatments appear to be on the horizon. Doctors are currently working to develop an inhaled version of the therapy that may also protect against the disease. And gene therapy is being studied as well. In this case, scientists are trying to find a way to put a normal copy of the Alpha-1 gene into a virus that has been made harmless to humans. The gene-carrying virus would then be administered to the patient, either by inhalation or other means, delivering the corrected copy of the gene to the tissues that need it. This therapy would be most beneficial to people early in life, before the disease damages the lungs and causes emphysema. Once damaged, the lungs cannot be restored to their previous condition.

Some patients with Alpha-1 have undergone a new procedure called lung volume reduction surgery, or LVRS. In this surgery, surgeons cut away the diseased portion of lung, thus allowing the healthy portion to work better. Results, however, have been mixed. Because most Alpha-1 patients have diffuse emphysema (as opposed to more localized emphysema in normal cases), many do not have enough healthy lung tissue left to take over for the diseased tissue that is cut out.

Alpha-1 patients diagnosed with end-stage lung disease may be candidates for a lung transplant. Doctors have reported some success with transplants, but long-term outcomes are lacking.

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