Acute myeloid leukaemia (AML) is a very aggressive and fatal form of cancer of white blood cells.  This year, an estimated 21,450 people of all ages (11,650 men and boys and 9,800 women and girls) in the United States will be diagnosed with AML, which makes it the second most common type of leukemia diagnosed in adults and children, but most cases occur in adults. 

Recently a study published in Cell Stem Cell, discovered that a protein, known as YTHDF2, is needed to trigger and sustain AML, but is not needed for healthy cells to function which opens several possibilities in terms of AML treatment.  This could aid the development of new drugs with significantly less harmful side effects than existing chemotherapy.

The study was headed jointly by a team of researchers led by University of Edinburgh and Queen Mary University of London and was done in collaboration as well with between the University of Manchester, Harvard Medical School and the Université de Tours; it was supported as well by Cancer Research UK and Wellcome.

Tests were done in blood samples donated by some leukaemia patients and in mice given AML.  Results of the study showed that the protein is abundant in cancer cells, while experiments in mice found that the protein is required to initiate and maintain the disease.  They further discovered that interfering with the biological pathway which in turn interferes with the function of YTHDF2 selectively kills blood cancer cells.

Another noteworthy discovery is that YTHDF2  is not needed to support the function of healthy blood stem cells, which are responsible for the production of all normal blood cells, and in fact were even more active in the protein's absence.

Professor Kamil Kranc, of Barts Cancer Institute, Queen Mary University of London, who jointly led the study, said: "Our work sets the stage for therapeutic targeting of cancer stem cells in leukaemia while enhancing the regenerative capacity of normal blood stem cells. We hope this will establish a new paradigm in cancer treatment."

Professor Dónal O'Carroll, of the University of Edinburgh's School of Biological Sciences, who co-led the research also said: "The study shows the promise of a novel class of drugs as the basis for cancer and regenerative medicine treatments."