A Device for the removal of circulating autophagic vesicles in patients with sickle cell disease (360G-Wellcome-201082_Z_16_Z)

Sickle Cell Disease(SCD) is the commonest inherited genetic disorder in the UK with 14,000 patients and over 300 affected children born annually. There are three disease modifying treatments available, hydroxyurea, blood transfusion and stem cell transplant. Of these, transfusion is the mainstay of preventative treatment. However, there are frequent clinical situations where transfusion would be recommended but blood is not available in sufficient quantity on a regular basis or not available at all. Ninety percent of patients with SCD worldwide live in the developing world where there is no safe and freely available access to blood. Consequently, there is a need for a novel therapy that does not require donated red cells. Professor David Anstee proposes a novel technology to address this need. Recent work in his laboratory at NHS Blood and Transplant in Bristol has shown that a critical step in the formation of normal red blood cells involves the removal of membranous vesicles containing waste products from immature red cells when the cells pass through the spleen. Patients with SCD lose the function of their spleen very early in life and as a consequence vesicles cannot be removed and remain in their blood. The vesicles that are retained in the peripheral circulation likely contribute to the pathology of SCD. Professor Anstee and colleagues are developing a device to selectively remove these vesicles from the blood of patients with SCD and anticipate that patients undergoing this treatment will enjoy a dramatic improvement in their quality of life.