Grant recipient

Jakob Werner Hansen

Deciphering Vexas Syndrome through a Clinical Trial and Translational Research
Grant amount: DKK 9,506,647

Jakob Werner Hansen says: “VEXAS is a newly discovered disease first described in December 2020. It is caused by an acquired mutation in the UBA1 gene in the hematopoietic stem cells. The mutation is found on the X-chromosome, so it is primarily elderly male individuals which are diagnosed with the disease. The syndrome is characterized by autoimmune symptoms, such as fever, skin rash and cytopenia which are debilitating for the patients and affecting both quality of life and affects overall survival. This proposal outlines our plan to conduct a clinical trial in the Nordic countries using the promising drug (azacitidine), which is not currently approved for the treatment of these patients. Furthermore, we will investigate the cells from the blood and bone marrow to get a better understanding of the disease, this combined effort will possibly both improve outcomes for patients and strengthen our knowledge about the molecular biology underlying the disease.”.

Jakob Werner Hansen is currently doing his specialist training at the Department of Hematology, Rigshospitalet. He says further: “The Clinical Emerging Investigator fellowship will allow me to build my own research group with focused on the VEXAS syndrome and continue my work as combined clinician and researcher”.

Jakob Werner Hansen
Department of Hematology, Rigshospitalet