AAT protein deficiency may seem like a rare disease at first glance, affecting only one in every 2,500 people worldwide. However, this means that AAT protein deficiency is observed in hundreds of thousands of individuals.
This genetic disease, if left untreated or improperly treated, has such a negative impact on the lungs and liver that it can lead to a fatal outcome.
The disease begins to develop in the liver and ultimately leads to chronic lung inflammation, causing the patient to slowly suffocate.
Treatment of AAT protein deficiency is quite complex; however, thanks to Israeli scientists, the situation may soon improve.
According to ISRAEL21c, the Israeli biopharmaceutical company Kamada, founded in 1990, has developed a "delivery" system for medications in patients suffering from the aforementioned disease, which is much more effective than the methods currently in use.
Today, this disease can be treated through liver and lung transplants or by continuous intravenous administration of AAT protein. The first option is not suitable for everyone, while the second requires weekly hospitalization and costs over one hundred thousand dollars a year.
Israelis have proposed an alternative treatment method – administering AAT protein not intravenously but through the respiratory tract.
By inhaling this protein, the need for constant repeated hospitalization disappears, and patients no longer waste time or experience discomfort. Additionally, the risk of infection, which is inevitable with the intravenous method, is eliminated.
Currently, the innovative method is under development and is undergoing primary and secondary phases of testing in Europe. These trials are expected to be completed in 2012.