Clues to precision treatments for a tough childhood leukemia

One of the biggest success stories in pediatric oncology is the effort to cure acute lymphoblastic leukemia (ALL). Survival rates for ALL, the most common childhood cancer, have risen above 90 percent. However, survival remains poor for children with treatment-resistant forms of the disease.

Enter a possible game-changer: A new study led by St. Jude on a tough-to-treat form of ALL called Ph-like ALL. In revealing genetic secrets of the disease, the study has identified promising treatment options in existing drugs.

Cancer cells in this form of ALL have a broken chromosome that gets put back together incorrectly. The study revealed not one, but four, similar rearrangements of the chromosome occurring in different patients. The rearrangements created a shortened version of a gene called EPOR. All led to the same thing: white blood cells proliferating out of control.

“To our knowledge, this is a previously unknown mechanism for leukemia,” said Charles Mullighan, MBBS, MD, of St. Jude Pathology.

Now for the good news: the researchers were able to counter the out-of-control biological processes triggered in Ph-ALL using an existing drug called ruxolitinib. In laboratory experiments, ruxolitinib also enhanced the function of other chemotherapy drugs.

“We think these findings provide a useful road map for planning more accurate testing of combination chemotherapies,” Mullighan said. In fact, one such clinical trial will take place at St. Jude.

The research was published in the journal Cancer Cell.

Read the news release