Sickle cell disease (SCD) is an inherited blood disorder characterized by the production of an altered form of haemoglobin in red blood cells. In sickle cell disease, haemoglobin polymerizes and becomes fibrous, causing red blood cells to become rigid and change form so that they appear ‘sickle-shaped’ instead of soft and rounded. Patients with sickle cell disease suffer from debilitating episodes of sickle cell crises, which occur when the rigid, adhesive and inflexible red blood cells block other blood vessels. Sickle cell crises cause excruciating pain as a result of insufficient oxygen being delivered to tissues, referred to as tissue ischemia, and inflammation. These events may lead to a variety of other adverse outcomes that require hospitalization.
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.
Sutimlimab is a first-in-class monoclonal antibody in development for the treatment of cold agglutinin disease, a rare form of autoimmune haemolytic anaemia, caused by cold-reacting autoantibodies. These antibodies bind to red blood cell membranes and destroy them, leading to anaemia. Symptoms include chronic debilitating fatigue, acrocyanosis, shortness of breath and other anaemia-related symptoms, leading to a poor quality of life and increased health resource utilization. In chronic cold agglutinin disease, the patient is more symptomatic during winter months. Therapeutic options may range from using warm clothing and avoiding exposure to cold weather to blood transfusions and chemotherapy.