Ponatinib is a targeted therapy medication used to treat certain types of leukemia. It works by blocking specific proteins that cause cancer cells to grow and multiply. This medication is taken orally and helps control the growth of cancer cells in patients with chronic myelogenous leukemia.

5-Azacitidine is a chemotherapy medication that affects how cells grow and divide. It is commonly used to treat blood disorders and certain types of blood cancers. This medication works by helping bone marrow produce normal blood cells and by killing abnormal cells in the blood. It is typically given as an injection under the skin or through an intravenous line.

The combination of these two medications is being studied to treat patients with chronic myelogenous leukemia that has progressed to more advanced stages (accelerated phase or blast crisis).

Chronic Myelogenous Leukemia – A type of blood cancer that begins in the bone marrow and affects blood cell production. The disease develops when bone marrow cells that produce white blood cells begin to grow and function abnormally. It typically progresses through three phases: chronic phase, accelerated phase, and blast crisis phase. In the accelerated phase, there is an increase in abnormal blood cells, and the disease becomes more aggressive. The blast crisis phase is the most advanced stage, where immature blood cells (blasts) multiply rapidly in the bone marrow and blood. During these advanced phases, the bone marrow produces excessive amounts of abnormal white blood cells that don’t mature properly.