Targeting fusion proteins to treat AML    

What if we could specifically target AML cells from the inside, whilst sparing healthy cells? Could this lead to transformative new treatments?



Professor Terry Rabbitts, Institute of Cancer Research, will explore a new approach to targeting cancer cells. This could lead to improved AML treatment with fewer side effects.

The challenge

Every year, almost 3,100 people in the UK are diagnosed with acute myeloid leukaemia (AML), the most common type of acute leukaemia. AML can progress very quickly and usually requires immediate treatment.

Leukaemia is caused by mutations in the DNA of normal cells, causing them to become cancerous. Sometimes chromosomes break and are joined to other chromosomes. If this happens it can result in fusion genes that code for cancer-causing fusion proteins. Because these fusions are cancer-specific they are ideal targets for treatment.

However, fusion proteins in leukaemia can be difficult to drug because, as Prof. Rabbitts highlighted in previous research, they have specific properties in controlling how proteins are made. New treatments need to avoid affecting the non-fused proteins that are in normal cells. This makes fusion proteins hard to turn off or remove.

The science behind the research

Professor Rabbitts and his research team want to explore a new approach to targeting fusion proteins – channelling antibodies inside cancer cells.

Antibodies are protective proteins produced by the immune system to help fight infection and are naturally found in the blood.  Antibodies have an exquisite natural selectivity so that they bind only  to one protein. This and their usual location in the blood stream is already exploited in many types of cancer treatment. The cancer-associated fusion proteins are inside cancer cells and not in the blood so natural, circulating antibodies are not useful.

Professor Rabbitts and his team have already developed a new technology that will allow them to deploy antibodies inside cells. His team will now develop antibodies that bind specifically to fusion proteins in AML and devise ways to get these inside leukaemia cells to remove the fusion proteins and cause the leukaemia cells to die.

What does this mean for patients?

This research is at an early stage, but will lead to the development of new AML treatments by binding antibodies specifically to cancer-specific fusion proteins inside cells, sparing normal cells.

Although future treatment regimens will require injections of these antibodies, the new approach will have fewer side effects, as it will focus on proteins that are only found in the cancer cells.

Professor Rabbitts anticipates that in the long term, this research will lead to a transformative new AML treatment, with scope for the new technologies to benefit other blood cancers, as well as other cancers more broadly.

Project information

Lead researcher

Professor Terry Rabbitts


Institute of Cancer Research

Blood cancer type


Award type

Project Grant

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