A team of scientists spotted a gene that is overexpressed in acute myeloid leukemia stem cells. Its existence, they believe, provides a target drug companies could use to develop better, more precise treatments for the rare and often fatal disease.
Ulrich Steidl and colleagues at Albert Einstein College of Medicine of Yeshiva University made the finding, which zeroes in on the HLX gene. Details are published in the journal Cancer Cell. In mouse models of AML, they found that the gene over-expresses in leukemia stem cells "at abnormally high levels." Subsequently, they found the same thing in a large number of AML patients. Out of 354 people suffering from acute myeloid leukemia whose HLX expression data they collected, 87% over-expressed HLX compared with healthier individuals. Even more ominous, the researchers looked at 601 AML patients who consistently had a worse chance of survival with the greater level of HLX expression.
This is where the research offers promise for drug developers. By using a specific technique to beat back the HLX expression in AML cells taken from mice and AML patients, researchers were able to suppress growth of the leukemia cells. In another test, researchers took AML cells from mice and humans, altered them so they didn't express HLX and transplanted them into healthy mice. They did the same procedure using unaltered AML cells, also with healthy mice. The rodents with AML cells altered to suppress HLX lived a lot longer. In essence, HLX could be the next big target in the quest to fight AML.
Let's be clear here. This research is in its early stages and years before major testing in humans, and there is no guarantee that the results will end the same in people. But the university is serious about advancing its finding into a viable treatment and says it wants to license the HLX technology.
The advance comes alongside mixed developments in the bid to come up with viable acute myeloid leukemia treatments. Stemline Therapeutics, which is struggling to advance an AML treatment, recently delayed its IPO though its drug is set to advance into Phase IIb trials.
On the other hand, other researchers are coming closer to better mapping how acute myeloid leukemia works, a crucial step toward creating targeted treatments. Scientists at the Genome Institute at Washington University in St. Louis recently determined that a few genetic "driver" mutations are all that are needed to bring on the disease. And folks are continuing to search for drugs that will kill the complicated and deadly form of cancer. Dana-Farber Cancer Institute scientists, for example, recently discovered a new peptide compound that appears to trigger enhanced tumor cell death in acute myeloid leukemia in mice, but largely left surrounding healthy tissue alone.
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