"Following this discovery, we wanted to test whether it could be used as a viable approach to treat leukemia in humans," adds Dr. Mry. "We transplanted cells from a patient with T-cell leukemia into a mouse. We then inhibited the Gfi1 molecule using a commercially-available agent, and noticed that it stopped the expansion of human leukemia in the bone marrow, peripheral blood and spleen, without leading to adverse effects."
"These results are a significant indication that therapies targeting the molecule Gfi1 would work in human patients," says Dr. H. Leighton Grimes, co-corresponding author of the study from the Cincinnati Children's Hospital Medical Center. "In fact, if our results translate to patients, they could improve the prognosis of people suffering from lymphoid malignancies," adds Dr. James Phelan, the study's co-first author and recent PhD graduate in Dr. Grimes' laboratory.
"Our study suggests that a molecular-based therapy targeting Gfi1 would not only significantly improve response rates, but may also lower effective doses of chemotherapy agents or radiation, thereby reducing harmful side effects," concludes Dr. Khandanpour, who is also a visiting scientist at the IRCM. "Gfi1 represents an Achilles' heel for lymphoid leukemia and we are continuing to work so that our approach may soon move to clinical trials."
|Contact: Julie Langelier|
Institut de recherches cliniques de Montreal