Crizanlizumab is a medicine under development for the preventative treatment of SCD painful crisis. Circulating platelets (type of cells in the blood that has a role in blood clotting and stopping of bleeding) in SCD patients express higher levels of P-selectin, a protein that drives the blockage of blood vessels. Crizanlizumab acts by blocking P-selectin and thus can prevent blockage in small blood vessels and maintain blood flow. Therefore, if licensed, crizanlizumab will offer a new treatment option for patients with sickle cell-related pain crises.
The amino acid L-glutamine has been developed as an oral powder formulation to reduce the acute complication of sickle cell disease. It is thought L-glutamine works by reducing cell inflammation and promote the cellular uptake of oxygen. If licensed, L-glutamine oral powder may offer an additional therapy option for those with sickle cell disease who currently have few effective therapies available.