Potential treatment for acute myeloid leukaemia identified

In collaboration with the Press Association

Scientists from the United States have identified a protein that affects the development of acute myeloid leukaemia (AML) and found that an experimental drug blocks its ability to cause cancer, according to research published in the journal Nature.

Experts from the Cold Spring Harbor Laboratory, and researchers from five other institutions, used a lab technique called RNAi screening to show that the protein Brd4 contributes to the development of AML.

This protein helps in the regulation of gene expression, which is the process by which information from a gene is passed on in order to create proteins - crucial to cell replication.

Specifically, the researchers found that Brd4 plays a part in deciding which genes are switched on in AML and how they work. And it is changes like these, in how genes are expressed, which contribute to the uncontrolled growth of cancer cells.

Following their initial work to pinpoint Brd4 as important factor in the development of AML, the researchers then tested an experimental drug already known to block this type of protein. They were able to suppress the disease in cells in the lab and model systems.

Team leader Dr Chris Vakoc, said: "The drug candidate not only displays remarkable anti-leukaemia activity in aggressive disease models and against cells derived from patients with diverse, genetic subtypes of AML, but is also minimally toxic to non-cancerous cells.

"The drug is currently being developed for therapeutic use for cancer patients and is expected to enter clinical trials within two years."

Oliver Childs, senior science information officer at Cancer Research UK, commented: "This is interesting research on a potential new way to treat AML, although still too early to benefit patients. Around one-third of all people diagnosed with leukaemia in the UK have AML. And despite improvements to treatment in recent years, there's still an urgent need for new ways to tackle this disease - to ensure that even more people survive.

"The next step will be to see whether these promising lab results can be successfully used to help treat people with AML."

The researchers are now planning to use a similar approach to discover drug targets in other types of cancer such as prostate and pancreatic cancers as well as melanoma.

Copyright Press Association 2011


  • Zuber, J. et al. RNAi screen identifies Brd4 as a therapeutic target in acute myeloid leukaemia. Nature doi:10.1038/nature10334