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Blocking essential genes to fight leukaemia

Blocking essential genes to fight leukaemia

Funding aims to focus attacks on harmful cells

Leukaemia is one of the most common types of childhood cancers and can often be cured.

However, some subtypes, like B-cell acute lymphoblastic leukaemia, can still be very dangerous for children. The treatment for this type of leukaemia is very intense, and patients can end up with more problems from the treatment than from the cancer itself.

Every researcher would agree that we need safer and more effective treatments. A key challenge is that while it is easy to get rid of cancer cells – researchers do it all the time in the  lab, when they drop their used equipment into bleach - our healthy cells are very easy to harm, too.

The difficulty comes in making sure that all of the cancer cells have died, without causing too much damage to healthy cells.

Dr Tariq Enver hopes the project will lead to new treatments.

In 2021, we funded a project from Dr Tariq Enver, who works at University College London. His research team had found that leukaemia has a weakness – the cancer cells need a gene called RUNX1 to survive.

When Tariq tested a chemical that blocked RUNX1 from working, he found that even high-risk types of leukaemia cells died – but the medicine did not harm healthy blood cells.

However, the chemical Tariq used could not be used for patients, because it isn’t strong or stable enough. In this project, his team have been trying to find a version of the chemical that could work for patients.

They’re looking at lots of different versions of potential medicines in the laboratory to find out which are the safest and most effective.

The researchers also want to give their new treatment the best chance of working by combining it with another medicine, which attacks the way in which leukaemia cells are addicted to RUNX1. They’ll be assessing all of the potential medicines together to find the best combinations.

Tariq hopes that this project will lead to new treatments that only harm leukaemia cells. This would help make sure more children live happy and healthy lives free from cancer and side effects.

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The MBE for voluntary groups was awarded to The Little Princess Trust by Her Majesty Queen Elizabeth.