U professor discovers new anti-leukemia gene

by Heather Fors

Dr. Catherine Verfaillie, a University associate professor of medicine, had originally gone to a sports university in Belgium to become a trainer or a physical therapist, but a knee injury during her first year forced her to choose a different area of study.
She chose to become a doctor, a decision with which she said she is now very happy. It is safe to say that leukemia patients nationwide will be just as happy she made the switch.
Verfaillie recently discovered an anti-leukemia gene that could possibly turn off the gene for chronic myelogenous leukemia.
A report detailing her discovery was published Dec. 15 in the journal Blood.
Currently, the only treatment for CML is to have a bone marrow transplant, she said. But the new gene developed by Verfaillie would give a safer alternative to patients who cannot have transplants. For patients over the age of 50, the chemotherapy and radiation needed for a transplant are generally too toxic, she added.
The new treatment would consist of removing thousands or even millions of CML cells from the body and replacing them with treated cells, which contain the anti-leukemia gene.
So far the gene has only been tested on mice, but with an injection of 1 million or more of the treated cells, researchers have seen a 100 percent survival rate among the diseased mice.
Verfaillie said the new anti-leukemia gene stops stem cells — found in bone marrow and the source of all blood cells — from producing leukemia and tumor-causing proteins. The gene also makes the stem cells resistant to chemotherapy, which will ensure that after they are transplanted back into the patient’s body, the cells will not be damaged from additional chemotherapy, she said.
However, for leukemia patient Larry Battis, a 59-year-old salesman from Burnsville, this treatment will come too late.
In August, after a yearly bone marrow test, Battis was told that he was back where he started almost six years ago. Because doctors don’t plan to begin testing the new anti-leukemia gene on humans until June, his only option for treatment is a transplant.
However, Battis is helping in the anti-leukemia gene research by agreeing to take a modifying gene that allows doctors to track the progress of his leukemia.
“I’m really the precursor … to what’s gonna happen in June,” Battis said.
Verfaillie said that CML is different from other cancers in that it is an infrequent disease, affecting only 7,000 new patients — mostly those aged 40 to 60 — every year.
She added that if the treatment of this particular type of leukemia is as successful in humans as it was in mice, doctors could have the basic key to turning off many forms of leukemia.