2019 participants
Alex Coburn
aHUS is an extremely rare genetic disorder which leads to kidney failure and low red blood cell counts. It is an immune system disorder caused by a mutation which affects the genes coding for proteins of the complement system, which makes up part of the innate immune system that protects us from pathogens. In people with aHUS the complement system is over active as it is the genes coding for inhibitory proteins, which usually keep the complement system under control, that are mutated. The project was focused on finding an alternative treatment to the standard drug Eculizumab, which is a poor drug due to its high cost and damaging side effects. This was done by testing monoclonal antibodies to find ones which can inhibit specific complement system proteins safely and effectively. A suitable compound was found; it will likely be used as a diagnostic lab tool in the future.
Funding source: Newcastle University
Project supervisor: Dr Kevin Marchbank