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.
BPX‐501 is a medicinal product made of T‐cells, a type of white blood cell, extracted from a donor who is partly matched to a patient undergoing HSCT. These T‐cells are expected to help the patient to fight off viral infections while their immune system is being restored with the transplanted stem cells. However, the transplanted T‐cells can cause GvHD. BPX‐501 incorporates a safety mechanism where the T‐cells are genetically modified to include a ‘suicide gene’. If the patient develops GvHD, a medicine called rimiducid is given to switch on the suicide gene in the T‐cells. This causes the T cells to die, thus preventing worsening of the GvHD. BPX‐501 has the potential to reduce hospital admissions, infections and improve survival when used in combination with standard care.