Venetoclax in combination with azacitidine for myelodysplastic syndrome

Venetoclax in combination with azacitidine is currently in clinical development for the treatment of myelodysplastic syndrome (MDS) in newly diagnosed higher-risk adults. MDS occurs when the blood-forming cells in the bone marrow (inner part of the bone that creates blood cells) become abnormal, leading to low numbers of one or more types of blood cells. These abnormal blood cells in MDS, either stay in the bone marrow or are destroyed before they get into the bloodstream. As the condition develops, the bone marrow becomes full. The immature blood cells then spill out into the bloodstream. The low numbers of normal blood cells in the bloodstream eventually cause symptoms such as reduced blood cell count (anaemia), feeling weak, tired, and breathless during activity, and repeated infections. High-risk MDS has the highest potential of developing acute myeloid leukaemia. There are currently limited treatment options for patients with high-risk MSD therefore there is a need to increase the treatment options available to this group of patients. Venetoclax blocks proteins BCL-2, which aid cancer cell growth/survival. By blocking this protein, it can kill and slow down the growth of cancer cells. Venetoclax is taken orally as a tablet, once daily. Venetoclax may delay this progression and thereby increase survival.