First in Europe to recruit patient to Sickle Cell Disease study

Congratulations to the adult non-malignant haematology research team at Manchester Royal Infirmary who have recruited the first patient in Europe to a phase III study for patients with sickle cell disease.

Led by Consultant Dr Ryan, the study aims to research the effect of study drug GBT440, in increasing red blood cells and reducing the severity of symptoms of sickle cell disease compared to the current treatment options available.

Sickle cell disease is an inherited disease, mainly affecting people of African and Caribbean origin. People with the disease produce red blood cells which have a tendency to go out of shape and become sickle-shaped (like a crescent moon)  instead of their normal disc shape. This means the cells can’t carry oxygen throughout the body which can cause severe pain and organ damage.

GBT440 is designed to work by helping haemoglobin, the molecules inside red blood cells, hold onto more oxygen as the red blood cells travel through the body. This keeps red blood cells in their normal shape and helps stop sickling.