Dr Konstantinos Tzelepis, a Leukaemia UK John Goldman Fellow, has developed a new drug that blocks a key leukaemia molecule called METTL3. This could lead to a new treatment that may give thousands of cancer patients a better chance of survival.

The challenge

Acute myeloid leukaemia (AML) is the most common type of leukaemia in the UK, but sadly the chances of survival from the disease are poor. Yet, despite decades of research, there have been very few advances in the treatment of the condition. We urgently need to new find treatments for AML and stop people dying from this disease.

The science behind the research

In previous research, Dr Konstantinos Tzelepis identified a molecule called METTL3 which is produced in large amounts in certain AML cells, and plays a key role in the development of leukaemia. He and his colleagues found that removing METTL3 killed AML cells, but did not affect normal blood production. This suggested that METTL3 could be a useful target for drugs to treat AML.

Since then, Konstantinos has been working with a pharmaceutical company who capitalised on his findings and have developed a small drug-like molecule which can block METTL3 – representing the first in a brand-new group of treatments.

In this John Goldman Fellowship project, Konstantinos’ plans confirmed that the METTL3 blocker drug could be an effective treatment for AML using animal models and cells donated by people with AML in order to study the particular anti-leukaemia effects the drug has.

What difference will this research make?

As Konstantinos’ project was successful, the METTL3 blocker has progressed into clinical trials to test its safety and effectiveness in human volunteers. This could eventually lead to a brand-new treatment for AML, and may also be effective for other cancers too. Ultimately, this important project could one day help to improve the chances of survival for thousands of people with this disease.