The first patient has been dosed at UCLH in a trial that could drastically improve the lives of people with a rare blood disorder.
In patients with thrombotic thrombocytopenic purpura (TTP), blood clots form in small blood vessels throughout the body. These clots can limit or block the flow of oxygen-rich blood to the body's organs and result in serious health problems such as strokes or epileptic events. TTP is caused by a lack of activity in the ADAMTS13 enzyme, a type of protein in the blood.
Participants in the trial will all receive IdeS, an enzyme that inactivates immunoglobulin G antibodies, to investigate if one dose will result in in increased ADAMTS13 activity. The study team, led by BRC supported Dr Marie Scully, will measure the effect by following the concentrations of the ADAMTS13 enzyme and antibodies to this enzyme as these are indicators of the progression of the disease.
In previous research in patients eligible for kidney transplantation, it has been shown that IdeS is highly effective in removing antibodies.
Dr Scully, consultant haematologist at UCLH, said: “This novel therapy, initially investigated to determine safety and efficacy, could provide an innovative treatment for acute TTP, clearing the antibodies against ADAMTS 13, aiding response to remission and reducing morbidity and mortality.”
The study is supported by Hansa Medical AB.
