According to The Guardian, England’s National Health Service (NHS) has announced that a new therapeutic option will be available for patients with sickle cell disease (SCD). This treatment, called crizanlizumab, is the first available SCD therapy in England over the last 20 years. Once this treatment becomes available, the NHS believes that it will help over 5,000 patients in the coming years.
Crizanlizumab
The National Cancer Institute (NCI) describes crizanlizumab as:
A humanized monoclonal immunoglobulin G1 anti-P-selectin antibody with vaso-protective and anti-vaso-occlusive properties. Upon administration, crizanlizumab binds to P-selectin and blocks its interaction with P-selectin glycoprotein ligand-1 (PSGL-1; SELPLG) on neutrophils and monocytes.
Let’s break that down a little. Basically, crizanlizumab works by blocking p-selectin. In turn, researchers believe that the drug prevents oxygen and blood restrictions, reduces pain crises, stops platelets from accumulating, and maintains adequate blood flow.
Currently, many patients – and doctors – are excited about crizanlizumab availability. While other treatments are available, such as hydroxycarbamide and blood transfusions, these are not always effective in severe cases of sickle cell disease. Thus, crizanlizumab could fulfill an unmet need within this patient population. The treatment will be available for patients aged 16+.
Sickle Cell Disease (SCD)
Sickle cell disease (SCD) consists of a group of inherited blood disorders characterized by malformed, sickle-shaped red blood cells. SCD results from genetic mutations, which cause the production of abnormal hemoglobin. These malformed cells then become stuck along the walls of blood vessels, causing blockages and restricted blood flow. Those of African American descent are at an increased risk of SCD. Symptoms usually appear in infancy, around ages 5-6 months. Since SCD is a lifelong condition, patients will experience symptoms throughout their lives. These include:
- Jaundice (yellowing of the skin and eyes)
- Delayed growth
- Retinal detachment and vision loss
- Gallstones
- Fatigue
- Swelling of the hands and feet
- Increase in urinary frequency
- Leg ulcers
- Anemia (low red blood cell count)
- Pain crisis
- Organ damage
