Scientists identify drugs to target ’Achilles heel’ of Chronic Myeloid Leukaemia cells

New research, by the Universities of Glasgow and Manchester, has revealed an 'Achilles heel' of Chronic Myeloid Leukaemia (CML) and found drugs to successfully target this weakness and eradicate the disease in mice. The study, which is published in Nature today, analysed both CML and normal blood stem cells and found two proteins that were key to the survival of CML stem cells. The group, which has been working on this research for more than six years, then developed a drug combination to simultaneously target these critical proteins and kill the cancer stem cells, while largely sparing normal cells. The interdisciplinary research team, led by Professor Tessa Holyoake from the University of Glasgow and Professor Tony Whetton from the University of Manchester, used a range of techniques to show that these two proteins (p53 and c-Myc) act as 'gateway controllers' in CML. Guided by the concept of precision medicine (the right drug, at the right time, for the right effect in the patient), the team designed a new treatment to exploit this critical weakness in the cancer. Using CML cells transplanted into mice, the authors demonstrated that drugs targeting these two proteins killed the cells that cause the leukaemia, effectively eradicating the disease. The results have potential implications for other cancers including acute myeloid leukaemia and brain tumours.