The treatment of patients with anaemia

Núria Nogués Gálvez

Banc de Sang i Teixits (BST), Barcelona, Spain

More than seven million people worldwide suffer from sickle cell anaemia, an inherited disorder of red blood cells that affects haemoglobin, the protein that carries oxygen to all organs and tissues in the body. The red blood cells of people with sickle cell disease are fragile and break easily, leading to chronic anaemia. In addition, they are not flexible or deformable like normal red blood cells, but rigid and crescent-shaped (falciform or sickle-shaped), which hinders blood flow and eventually clogs the narrowest vessels (the capillaries). When this happens, there is a lack of oxygen to the tissues, causing pain and organ damage.

Blood transfusions are an essential part of the treatment of this disease, which can only be cured, for the time being, through a bone marrow transplant. Sickle cell anaemia mostly affects people of African descent, although migratory movements mean that the number of patients with the disease has also been steadily increasing in Spain. This poses a significant challenge, especially due to the lack of compatible blood for patients with rare blood groups, as most blood donors are of Caucasian origin.

To address this problem, work is underway on the in vitro production of red blood cells and, although the process still has many limitations, significant progress has recently been made. The aim of this project is to establish a renewable source of red blood cells with blood group profiles that are severely under-represented in the European donor population. To this end, precursor cell lines will be generated from selected donors with relevant rare blood groups found in African populations, and an efficient and scalable production system will be designed to overcome the limited availability of compatible blood for treating patients with this disease.

• Jan Frayne, University of Bristol, United Kingdom

Lab-grown red cells for the transfusion management of vaso-occlusive crisis in sickle cell patients

€941,350.12