A new genetic target has been found for potential treatments for acute myeloid leukaemia, British researchers have reported in Nature.
Scientists at the Wellcome Trust Sanger Institute, Hinxton, Cambridge, focused on the activity of a gene called METTL3, which they identified in genetic screening as essential for the growth of acute myeloid leukaemia. They discovered a pathway specific to METTL3 which helps maintain the cells.
The scientists state that: "Together, these data define METTL3 as a regulator of a chromatin-based pathway that is necessary for maintenance of the leukaemic state and identify this enzyme as a potential therapeutic target for acute myeloid leukaemia".
They showed in vitro that inhibiting METTL3 destroys human and mouse acute myeloid leukaemia cells without harming non-leukaemic blood cells.
The team hope that this unexpected new drug target could open new avenues to develop effective treatments.
Project leader Professor Tony Kouzarides, from Cambridge University’s Gurdon Institute, said: "New treatments for acute myeloid leukaemia are desperately needed and we have been looking for genes that would be good drug targets. We identified the methyl transferase enzyme METTL3 as a highly viable target against acute myeloid leukaemia.
"Our study will inspire pharmaceutical efforts to find drugs that specifically inhibit METTL3 to treat acute myeloid leukaemia."
Fellow researcher Dr Konstantinos Tzelepis, from the Sanger Institute, added: "This study uncovered an entirely new mechanism of gene regulation in acute myeloid leukaemia that operates through modifications of RNA. We discovered that inhibiting the methyl transferase activity of METTL3 would stop the translation of a whole set of proteins that the leukaemia needs."
Haematologist Dr George Vassiliou, from the Cambridge University Hospitals NHS Trust, said: “We believed that we had to think differently and look in new places for ways to treat the disease and in METTL3 we have found an exciting new target for drugs.
“We hope that this discovery will lead to more effective treatments that will improve the survival and the quality of life of patients with AML.”
Source: Barbieri, I. et al. Promoter-bound METTL3 maintains myeloid leukaemia by m6A-dependent translation control. Nature 27 November 2017 doi: 10.1038/nature24678
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