.jpg)
DRUG REPOSITIONING FOR THE TREATMENT OF ANEMIAS
EP23382485.3
The present proposal focuses on the use of ZAKα (MAP3K20) kinase inhibitors, or a composition comprising them, for use in the treatment of anemia. These are compounds already employed in other types of applications, which have been shown to inhibit a specific inflammasomal pathway involved in the regulation of hematopoiesis. Specifically, they promote erythropoiesis (red blood cell production). To this end, assays have been carried out using both human cells and the zebrafish animal model. The proposal is based on the use of ZAKα kinase inhibitors to alter a signaling pathway, specifically ZAKα/P38/NLRP1, which promotes the accumulation of the erythroid transcription factor GATA1 and, therefore, erythropoiesis. Studies have demonstrated its therapeutic efficacy in the treatment of different types of anemias, especially congenital anemias or anemias associated with chronic inflammatory diseases. More specifically, the results obtained with hematopoietic progenitors from Diamond Blackfan anemia patients show a very clear therapeutic benefit in patients with different mutations. This proposal includes an in vitro method to screen, identify and/or produce compounds for use in the treatment of anemia.
- Identification of different compounds that have in common the regulation by inhibition of a new specific signaling pathway involved in hematopoiesis. - Repositioning of drugs for the treatment of rare anemias for which there are still no effective therapeutic options. The invention is particularly interesting for companies in the medical and pharmaceutical sector, as it reveals new therapeutic strategies for the treatment of hematopoietic disorders associated with chronic inflammatory diseases and congenital anemias, providing an answer to a medical need not covered by current treatments. This shows the clinical relevance of these findings for treating anemia, particularly congenital anemia or anemia associated with chronic inflammation.