Eculizumab, a monoclonal antibody targeting and preventing cleavage of C5, is FDA approved for only four rare conditions. However, complement is suggested to play a key role in numerous other disease entities and during the last 10 years a growing medical knowledge, faith and willingness to adopt complement inhibitory therapy to other patients groups is evident.
This project aims to reveal novel insight into pathophysiological processes of different human diseases where complement plays a key role. Antiphospholipid syndrome, sickle cell disease, and microangiopathic disorders of different origins are examples of conditions where complement may play a crucial pathogenic role. Randomized clinical trials will for most of these diseases often not be possible because they are rare and featured by a complex heterogeneity. Thus, case series and case reports definitely represent a valuable contribution to new and important knowledge. In this setting, a profound and targeted examination of selected patients inflammatory profile is pivotal and hypothesis generating. When possible, ex vivo models mimicking the specific diseases will be established, enabling better understanding of the complement pathophysiological mechanistic.
Connecting basal science closer to the clinic is part of translational research and is one of the most important challenges for todays and future medicine. This project aims to meet this challenge and has a clear advantage by hosting scientists working both in the lab and in the clinic with critical ill patients.