Since only the lung involvement in AAT deficiency could be influenced therapeutically, several studies are now planned or already being carried out in which the effects of AAT deficiency on the liver should be (co-)treated. Current studies are currently only concerned with subjects of the genotype Pi*ZZ.
On the one hand, studies by Arrowhead Pharmaceuticals are underway, in which the production of AAT is suppressed by means of siRNA (small interfering RNA). Through the inhibited formation of the liver toxic protein, the liver should be relieved for up to two years and be able to regenerate. The therapy does not target the genetic material (i. e. DNA), but only short-lived copies of DNA (the so-called mRNA) are addressed. The intended long-term treatment is checked by means of a liver biopsy. The two studies AROAAT2001 and AROAAT2002 are already fully recruited. Initial evaluations show promising results.
- AROAAT2001: fully recruited
- AROAAT 2002: fully recruited
On the other hand, a study by Dicerna in Germany is currently starting. This study also uses siRNA, which is given to patients with alpha1 antitrypsin deficiency at a certain stage of fibrosis.
- Dicerna: currently starting
Careful consideration of inclusion and exclusion criteria is required for the respective studies. If you have any further questions about the studies or would like further information, please do not hesitate to contact us.