A physician and researcher affiliated with The Jackson Laboratory in Bar Harbor says he may have identified a cure for the most common form of human leukemia.
Dr. Shaoguang Li, now conducting research at the University of Massachusetts Medical School in Worcester, has identified a specific gene that supports the development of chronic myeloid leukemia, as well as a drug treatment that targets that gene to short-circuit the proliferation of leukemic blood cells.
In a telephone interview Tuesday, Li said the results of his study, published in the current issue of the journal Natural Genetics, is good news for victims of chronic myeloid leukemia.
“The power of this strategy is for curing the disease, not just controlling it,” Li said.
The gene, called Alox5, allows leukemia-producing stem cells to develop and proliferate, Li said.
In research at The Jackson Laboratory, Li studied mice that had been specially bred to lack the Alox5 gene.
“If you remove the gene from a mouse, you don’t see leukemia develop,” he said. That’s because Alox5 is somehow tied to the development of cancer stem cells, the precursors to leukemia, he said.
Li also studied normal mice with leukemia, targeting the Alox5 gene with the drug Zileuton, which is approved for treating asthma. Zileuton successfully blocked the gene’s production of an enzyme that turns cancer stem cells into full-blown leukemia cells.
“When you block the gene’s function by using the drug … you’re going to be leukemia-free,” Li said.
Mice treated with a combination of Zileuton and Gleevec, the most effective treatment currently available for chronic myeloid leukemia, fared even better than mice treated with either medication alone.
Li said Zileuton must now be studied in human clinical trials before it can be prescribed for leukemia patients.
“A lot of patients will be interested” in participating in those trials, he forecast. “This is for a cure.”
Already, he said, he has been contacted by the father of a young leukemia patient about participating in a clinical trial. The drug’s current approval for use in treating asthma should fast-track the clinical studies, he said, but he declined to speculate on a timeline.
Li said there are larger implications as well.
“Now we know we can target cancer stem cells without damaging other stem cells,” he said. “Without this gene, normal blood can be produced, but the leukemia disappears.”
In a news release issued earlier this week, The Jackson Laboratory said it is seeking “patent protection” on the treatment developed by Li and his research colleagues at the University of Massachusetts Medical Center and the Dana-Farber Cancer Institute at the Harvard Medical School.
Li, who retains adjunct professor status at The Jackson Laboratory, said the Bar Harbor lab could “benefit a great deal” from his research findings.