The docking of two proteins that cause acute myeloid leukemia could pave the way for development of a new drug to fight the disease, as well as other types of leukemia and possibly other cancers.
A team of researchers from the University of Queensland has discovered a molecular target that could result in the development of a new drug that could be used to battle acute myeloid leukemia (AML). What’s more, the researchers believe that if such a drug were developed, it might also be effective in the treatment of other forms of leukemia, and possibly different kinds of cancer. The research findings appear in the journal Blood.
AML starts in the bone marrow, which is the soft inner part of the bones, where new blood cells are made. In most cases, it quickly moves into the blood. It can sometimes spread to other parts of the body, including the lymph nodes, liver, spleen, central nervous system (brain and spinal cord), and testicles.
The American Cancer Society (ACS) estimates that in 2014, there will be about 52,380 new cases of all kinds of leukemia and 24,090 deaths from all kinds of leukemia. The ACS expects about 18,860 new cases of AML, most of which will be in adults. The ACS also estimates there will be about 10,460 deaths from AML. Again, almost all cases will be in adults.
The researchers found that the “docking” of one protein, called the Myb protein, with another protein, the p300 protein, was critical for the development of AML. Senior researcher, professor Tom Gonda of the University of Queensland School of Pharmacy, said in a press release that the Myb protein was produced by the MYB oncogene, a gene that had the potential to cause cancer and which was required for the continued growth of leukemia cells. “Our data identifies the critical role of this Myb-p300 interaction and shows that the disruption of this interaction could lead to a potential therapeutic strategy,” said Gonda.
Gonda went on to say that the research could lead the way for development of a drug to block this interaction and stop the growth of AML cells, as well as the cells of other types of leukemia.
Cautioning that MYB is also essential for normal blood cell formation, Gonda said an approach for targeting it that won’t completely disrupt normal blood cell production is needed.
The researchers’ work demonstrates that normal blood cells can continue to form even when the Myb-p300 interaction is unable to occur. This finding suggests that a drug that blocks the interaction could be safe for use in patients, according to the researchers.
Emphasizing that the research is at a very early stage, Gonda said the researchers believe their findings have the potential to result in huge advantages in the fight against leukemia and, possibly, other cancers.
Since Myb was not a conventional target for drugs, the team may study other ways to target MYB, including targeting the genes and proteins that work downstream of MYB.
“If we can block the downstream molecules that are controlled by MYB, we may end up with the same result,” said Gonda.
Finally, Gonda said, “Drug development and subsequent clinical trials are long processes, but we are hopeful this research has a promising future.”