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UK scientists have helped show that a pair of genes inside cells could play a major role in how a serious form of leukaemia develops. The discovery opens the potential for new treatments that target the genes – which are known as KDM4C and PRMT1.
Researchers based at King’s College London, working with colleagues in the US and Hong Kong, found that the genes work together during the development of acute myeloid leukaemia (AML), changing healthy blood cells into cancer cells.
Funded by Bloodwise and Cancer Research UK, the early studies in mice offer hope that using drugs which block these genes could offer a new treatment.
Dr Áine McCarthy, science information officer at Cancer Research UK, said: “Identifying weaknesses in acute myeloid leukaemia cells is the key to developing much needed new treatments for the disease – and to helping more people survive. This exciting research has found a new way to target this disease and we look forward to seeing whether this could help patients in the future.”
In this study, published in the journal Cancer Cell, the two genes were either turned off inside cells using genetic tools, or experimental drugs were used to block their activity in mice with a form of leukaemia, to see if doing so helped the mice survive longer.
The researchers found that the majority of mice whose cells had either gene silenced were still alive at the end of the 60-day experiment. In contrast, the majority died in under 40 days with no treatment. Blocking the activity of either gene with drugs also extended the mice’s survival time.
Current treatment for the 2,800 UK patients diagnosed with AML each year involves aggressive chemotherapy. But only one in five patients survive for five years or more, and treatment can often be too harsh for older patients.
Professor Eric So, research leader at King’s College London, said: “The demonstration of how critical these genes are to cancer transformation could be highly significant for the design of new drugs. Further work is needed to develop and refine drugs to maximise their effects and so that they are suitable for patients. Clinical trials will then be needed to see how leukaemia patients respond to these drugs and how use of them can be optimised.”