An experimental drug called CYT387 blocks an enzyme that causes bone marrow cancers called myeloproliferative disorders (MPDs), according to U.S. researchers who conducted tests in mice and human cells.
The findings were to be presented Tuesday at the annual meeting of the American Society of Hematology, in San Francisco.
The researchers, from Oregon Health & Science University's Knight Cancer Institute, found that CYT387 was "very effective" against a specific type of cancer cell driven by an enzyme mutation called JAK2-V617F. The drug binds to the V617F mutation in the JAK2 enzyme.
"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," researcher Dr. Thomas Bumm said in a university news release.
There are no U.S. Food and Drug Administration-approved targeted treatments for the "big three" MPDs -- polycythemia vera, essential thrombocythemia and primary myelofibrosis.
"Based on the efficacy we demonstrated in the mouse model, there is a good chance that CYT387 will enter clinical trials as early as 2009," principal investigator Dr. Michael Deininger, head of the hematological malignancies program, said in the news release.
The study was funded by Cytopia Research Pty Ltd. of Australia, which makes the drug.
More information
The Leukemia and Lymphoma Society has more about MPDs.
SOURCE: Oregon Health & Science University, news release, Dec. 9, 2008
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