This technology is a new therapeutic method for Myelodysplastic syndrome (MDS) patients that increases platelet counts by reducing β-catenin protein levels.
Myelodysplastic syndromes (MDS) are a group of disorders characterized by the failure of hematopoietic stem cells to fully differentiate into mature blood cells. This results in low blood cell counts and a predisposition to the development of leukemia. The current standard of care for MDS involves frequent blood transfusions to improve low blood cells counts. However, repeated transfusions have significant clinical and economic drawbacks. Therefore, a better pharmacological treatment for MDS is needed.
This technology treats MDS patients with thrombocytopenia using all-trans retinoic acid (ATRA), which inhibits β-catenin expression and regulates differentiation of osteoblasts, resulting in improved and sustained platelet counts. This platelet stability is dependent on continued ATRA treatment, as withholding ATRA results in declining platelet numbers. Additionally, this technology identifies β-catenin signaling as a promising target for the treatment of MDS-related cytopenias, as activated β-catenin expression is elevated in osteoblasts and CD34+ cells prior to ATRA therapy, but not during treatment. One MDS patient with thrombocytopenia was successfully treated with an approved dose of ATRA, no longer requires transfusions, and is free of disease-related symptoms.
This technology is being investigated in other MDS patients who exhibited activation of β-catenin in their osteoblasts. Preliminary data shows stabilization of the disease and improvement of platelet counts after ATRA treatment.
Patent Pending
IR CU17377
Licensing Contact: Joan Martinez