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A step closer to effective leukaemia treatment

Scientists have reported a novel cancer treatment approach that could lead to a new drug for acute myeloid leukaemia (AML). The new approach targets enzymes connected with the development and maintenance of the disease.

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A recent study on AML is offering new hope for sufferers of the disease. AML is a rare type of blood cancer in which abnormal white blood cells build up in the bone marrow and quickly spread to the blood. Most common in people over 65 years of age, this life-threatening disease progresses rapidly and can lead to serious infections and organ failure if left untreated. In their study, researchers supported by the EU-funded LeukaemiaTargeted and CRIPTON projects have now presented a novel approach that could lead to a new class of drug for the treatment of AML. Their approach targets enzymes that play an important role in initiating and maintaining the disease. The results have been published in the journal ‘Nature’.

The role enzymes play

Our body needs proteins to keep working properly, but to create proteins its DNA must first be converted to ribonucleic acid (RNA). Enzymes carry out most of the chemical reactions that take place in cells, and they also affect the production of proteins by making chemical changes to RNA. However, sometimes enzymes become dysregulated and are produced in excessive quantities. In the case of AML, the enzyme METTL3 plays a role in the disease’s development when it’s overexpressed in certain cell types. Led by Prof. Tony Kouzarides of CRIPTON project coordinator University of Cambridge, the research team has now identified a molecule called STM2457 that can inhibit METTL3. “Proteins are essential for our bodies to function and are produced by a process that involves translating our DNA into RNA using enzymes. Sometimes, this process can go awry with potentially devastating consequences for human health. Until now, no one has targeted this essential process as a way of fighting cancer. This is the beginning of a new era for cancer therapeutics,” observed Prof. Kouzarides in a news item posted on the ‘Mirage News’ website. The researchers tested the effect of STM2457 in tissue cultured from human AML patients and in mice models of the disease. They found that treatment with the molecule led to reduced growth of cancerous cells and an increase in apoptosis, or the death of these cells. In the mice, STM2457 reduced the number of cancerous cells in the bone marrow and spleen without affecting body weight and with no toxic side effects. “This is a brand-new field of research for cancer and the first drug-like molecule of its type to be developed. Its success at killing leukaemia cells and prolonging the lifespans of our mice is very promising and we hope to begin clinical trials to test successor molecules in patients as early as next year,” stated co-author Dr Konstantinos Tzelepis, also of the University of Cambridge, in the same news item. According to Dr Tzelepis, this approach could also be used to treat many other types of cancer besides AML, offering the medical community “a new weapon in our arsenal against these terrible diseases.” The LeukaemiaTargeted (Selecting genetic lesions with essential function for patients’ leukaemia in vivo as targets for precision medicine) project is hosted by Helmholtz Zentrum München, German Research Center for Environmental Health. The 5-year project ends in July 2021. CRIPTON (Role of ncRNAs in Chromatin and Transcription) ended in 2017. For more information, please see: LeukaemiaTargeted project CRIPTON project


LeukaemiaTargeted, CRIPTON, cancer, leukaemia, AML, acute myeloid leukaemia, enzyme

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