New drugs against anaemia Two billion people suffer from red blood cell deficiency, or anaemia. Efforts towards new treatments are thus urgently needed. Fundamental Research Health © ducu59us, Shutterstock Traditionally, anaemia has been treated with recombinant erythropoietin (Epo) to increase red blood cell production. However, aplastic anaemia and secondary anaemia associated with cancer, inflammation and infection do not respond well to recombinant Epo treatment. As a result, there is a great need for novel treatments against Epo-resistant anaemias. Towards this goal, scientists on the EU-funded ERYTHROTHERAPY (Defining new drugs and drug targets for treatment of anemia) project set out to identify novel drug targets. In this context, they performed mRNA sequencing, identifying 250 candidate genes that have the potential to be targeted by drugs. Subsequent characterisation of these genes helped researchers to unveil novel factors that regulate red blood cell fate and promote red blood cell progenitor proliferation. This work will serve as the basis for erythroid cell reprogramming through a novel class of erythropoiesis-stimulating agents able to manage conditions that today lack pharmacological treatment. In another part of the project, researchers investigated the pathophysiology of Diamond-Blackfan anaemia (DBA), a congenital hypoplastic anaemia that is associated with reduced life quality and expectancy. The consortium generated a novel animal model for DBA to identify the underlying molecular mechanisms and determine mechanism-based treatment strategies. Given that nearly half of DBA cases are tied to abnormal ribosomal protein genes, scientists investigated the link between ribosomal protein deficiency, anaemia and bone marrow failure. They identified promising drug targets and candidates for DBA. Overall, the ERYTHROTHERAPY consortium took all the necessary steps towards developing anaemia-specific drugs. In addition, the new mouse model is expected to fuel future research into DBA treatment, including cell-based approaches. Keywords Anaemia, erythropoietin, ERYTHROTHERAPY, drug targets, Diamond-Blackfan anaemia
