Research Highlights

Combination treatment improves outcome for a fatal type of leukaemia 

Published online 7 April 2015

Combined RA/arsenic treatment reduces abnormal white blood cells in acute myeloid leukaemia patients.

Aisha El-Awady

A new study shows that adding a combination of retinoic acid (RA) and arsenic to chemotherapy in acute myeloid leukaemia (AML) patients could improve their chances of survival.  

Acute myeloid leukaemia is a cancer of the bone marrow that prevents blood-forming cells from normal maturation. If left untreated, AML can quickly become fatal. The most frequently mutated gene in AML is the nucleophosmin 1 (NPM1) gene. 

A team of researchers from Lebanon and France have found that, in NPM1-associated AML, combined RA/arsenic treatment significantly reduced abnormal immature white blood cells in some AML patients, publishing their findings in Blood1

Using AML cell lines or primary samples, the researchers discovered that the combined treatment causes degradation of the mutant NPM1, which leads to the arrested growth and programmed cell death of the AML cells. 

The NPM-1 mutation also causes promyelocytic leukaemia (PML) nuclear bodies to become disorganized. The bodies normally have a role in inducing programmed cell death, and the combined treatment restored them to their normal place within the nucleus. 

“Collectively, these results indicate that NPM1 mutant protein is a target of RA and arsenic treatment, providing a rationale for further exploiting these two drugs in the clinic for the treatment of NPM1-mutated AML,” says Bazarbachi.


El Hajj, H. et al. Retinoic acid and arsenic trioxide trigger degradation of mutated NPM-1 resulting in apoptosis of AML cells. Blood (2015).