PAI-1, hematopoietic stem cell, and chronic myeloid leukemia

The new treatment goal for chronic myeloid leukemia (CML) is treatment-free remission, which requires the achievement and maintenance of a deep molecular response (DMR) using a tyrosine kinase inhibitor (TKI). However, only a limited number of patients achieve or maintain DMR. TM5614, a PAI1 inhibitor, can bring quiescent hematopoietic stem cells from the bone marrow niche into the cell cycle. Thus, the combination of TM5614 and TKI may eradicate CML cells. Currently, a phase III clinical trial is confirming the efficacy of TM5614 and TKI combination therapy.