AML is a disorder due to the unbalance between proliferation and differentiation of the hematopoietic stem cells leading to an abnormal increase of immature myeloid precursors (blasts) in the bone marrow and in other organs and tissues such as spleen, liver and central nervous system. 

AML represents about 1.3% of all cancers in adult life.  Every year 4.2/100000 new cases are diagnosed, with a maximum incidence in the 60-65 age group. If untreated, the AML is quickly fatal. The therapy is however aggressive and tends to induce cell death and/or blast maturation.

The prognosis is unfavourable and patients  survival after 5 years is about 27%. To find innovative diagnostic and therapeutic tools, it is therefore important to dissect the molecular mechanisms governing proliferation and differentiation of the leukemic blasts.

A research team supervised by prof. Rosa Sorrentino at Sapienza, together with “University of Bari”, the “Bambino Gesù Children Hospital” and “Gemelli Hospital” in Rome, has identified novel molecular markers in AML. The study has focused on the activity of two enzymes, ADAR1 (ADAR) e ADAR2 (ADARB1), acting on the RNA molecules and leading to variations in gene expression both at quantitative as well as at qualitative level.

The team has shown that, during in vitro differentiation of the leukemic blasts, the enzymes are expressed with a different kinetics: ADAR1 is present in the leukemic blasts  where it is likely to play a relevant role for their survival whereas ADAR2 is eliminated during the proliferative phase and emerges when the blasts are differentiated.

Therefore, the two proteins mark the different phases of the blasts maturation thus lending themselves to be used in the clinic to monitor the blasts status and, eventually, to open the venue for innovative therapeutic strategies.