Oregon Health & Science University Knight Cancer Institute researchers have found that an experimental drug successfully blocks an enzyme that causes some bone marrow cancers.
The oral drug, called CYT387, was tested in mice as well as in human cells. In both cases, it blocked the growth of certain bone marrow cancers called myeloproliferative disorders, also referred to as MPDs.
The research will be presented Tuesday, Dec. 9, at 7 a.m. during the 50th annual American Society of Hematology conference in San Francisco.
"The drug was found to be very effective against a specific type of cancer cells, cancer cells which are driven by an enzyme mutation called JAK2-V617F. In the mouse model, the drug blocked JAK2-V617F, normalized blood counts and reduced enlarged spleens back to a normal size. It is a very promising compound," said Thomas Bumm, M.D., Ph.D., a member of the research team.
The drug works by binding to the V617F mutation in the JAK2 enzyme. Without this drug, the mutated JAK2 enzyme leads to MPDs. The "big three" MPDs include polycythemia vera, essential thrombocythemia and primary myelofibrosis. Until now there have been no FDA-approved targeted treatments for these diseases.