A study from the research team of Linda Smit shows promising results in targeting acute myeloid leukemia (AML) stem cells using a drug directed to two proteins involved in the regulation of transcription by chromatin modulation.

The research group of Linda Smit at the department of Hematology is developing treatment strategies to increase survival chances of AML patients. AML is a disease characterized by transcriptional dysregulation resulting in a block in differentiation and aberrant self-renewal. Chemotherapy is the standard treatment for AML patients, but unfortunately, after treatment often a small population of leukemia cells survives and is able to initiate recurrence, so-called leukemia stem cells. An inhibitor targeting both bromodomain and extraterminal domain (BET) and CPB/P300 proteins, named NEO2734, is efficiently reducing the viability of AML cells by inducing apoptosis, but is also able to eliminate leukemic stem/progenitor cells from AML patient samples. Importantly, NEO2734 could increase the effectiveness of combination chemotherapy treatment in an in vivo AML patient-derived xenograft mouse model. Thus, dual inhibition of BET and CBP/P300 is a promising therapeutic strategy for AML patients. The results of this research is published in the journal ‘Hemasphere