What is AML?

Acute myeloid leukemia or acute myelogenous leukemia (AML) is an aggressive type of blood cancer characterized by overproduction and accumulation of cancerous, immature white blood cells, also known as myeloid blast cells or leukemic blasts [1] (Figure 1). The accumulation of these abnormal white blood cells in the bone marrow affects the synthesis of normal blood cells, i.e. red blood cells, platelets and normal white blood cells, causing a decrease in all of them to varying degree [1]. Every year, AML affect around 20,000 new people in the United States with a median age of 67 years [1, 2].

Figure 1: Schematic of normal blood cells an AML development steps

How is AML treated?

Standard treatment for AML consists of two phases, namely (1) induction of remission and (2) consolidation [3, 4]. During the induction phase, the patient receives intensive chemotherapy to remove leukemic cells. However, a small number of leukemic cells, undetectable in blood or bone marrow tests, may survive and leukemia is likely to come back. Consolidation therapy aims at stopping this with more chemotherapy and, if the patient is suitable, with a bone marrow or stem cells transplant to replace leukemic stem cells with healthy ones.

Overall, 50 to 75% of AML patients will achieve remission after initial therapy, and from those, most will relapse resulting in short life expectancy [1, 3, 4]. Treatment options for patients that do not respond or experience relapse after initial therapy include different chemotherapy regimens, but these rarely result in long-term survival [3, 4].

Despite the advances in therapeutics and supportive care during the last decades, the survival rates of this malignancy remain low, thus the unmet medical need for new therapies is very high.


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