A NAC for TTP

U.S. and Canadian researchers have found that the generic drug N-acetylcysteine could be repurposed for use in thrombotic thrombocytopenic purpura, a rare hematological disorder for which cumbersome plasma exchange therapy has been the standard of care for almost two decades.¹ The group is seeking a partner to sponsor clinical trials in the new indication.

Activated endothelial cells produce and secrete long polymers of von Willebrand factor (VWF)that are linked together with disulfide bonds. These long VWF polymers adhere to platelets and collagen, which can trigger thrombus formation...