Study participants sought: Patients with repeatedly elevated liver enzyme levels and/or other indications of liver disease
Author
Priv.-Doz. Dr. med. Pavel Strnad – Head of the Alpha1 Liver Center and Scientific Advisory Board Alpha1-Deutschland eV &
Dr. med. Karim Hamesch – Coordinating Study Physician
Dear members and interested parties,
While previously only lung involvement in AAT deficiency could be therapeutically influenced, two studies are now being planned or are already in their initial phase, aiming to (co-)treat the effects of AAT deficiency on the liver. Both studies currently only include subjects with the PiZZ genotype.
Firstly, a study by the company Arrowhead is currently underway, in which the production of AAT is suppressed using siRNA. By halting the production of this hepatotoxic protein, the liver should be relieved of stress for up to two years and allowed to regenerate. The therapy does not target the genetic material (i.e., the DNA); it only addresses short-lived copies of the DNA (the so-called mRNA).
Secondly, a study by the company Vertex will begin in Germany in the coming months. This study aims to enable the secretion of the mutated AAT from liver cells into the bloodstream. For this purpose, participants will take one tablet daily for one month.
As previously mentioned, the Arrowhead study offers a longer-term treatment whose effectiveness should be verified by a liver biopsy, while the Vertex study will primarily examine changes in AAT levels in the blood. Therefore, the Arrowhead study is seeking participants with primary liver involvement, whereas the Vertex study will include most PiZZ participants who have not yet received augmentation therapy.
Should you have any further questions about the studies or require additional information, please feel free to contact us. We can be reached at the following telephone number: 0241-80 36606/35324
