The National Cancer Institute awarded a $378,674 Project Grant (CFDA 93.395 - Cancer Treatment Research) to The University of Texas MD Anderson Cancer Center (MD Anderson) to develop a small-molecule inhibitor of the EIF2AK1 protein kinase. The goal is to overcome ineffective erythropoiesis, or impaired red blood cell production, in patients with myelodysplastic syndromes (MDS) characterized by mutations in the SF3B1 gene. This novel therapeutic approach aims to address the severe anemia and high transfusion burden experienced by many MDS-RS (with ringed sideroblasts) patients, for whom current treatments like luspatercept have limited efficacy. The award supports preclinical studies to functionally validate candidate EIF2AK1 inhibitors using cellular assays with primary cells, induced pluripotent stem cells, and engineered cell lines developed by the research team at MD Anderson. If successful, this work could lead to the first EIF2AK1 inhibitor treatment for transfusion-dependent MDS-RS patients.
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