Genetic change reveals precision medicine possibilities for a high-risk leukemia

Acute lymphoblastic leukemia (ALL) is the most common childhood cancer and one of the most treatable. At St. Jude Children’s Research Hospital, more than 90% of patients with ALL are now cured. But sometimes therapies still fall short. For about 30% of ALL cases, the genetic basis remains unknown.

St. Jude scientists led an international team of researchers that recently filled some of those gaps. The findings include a promising lead on precision medicine for patients with a newly identified, high-risk form of ALL.

Researchers studied genetic material from more than 1,700 ALL patients. They were looking for new chromosome rearrangements that drive the development of disease. The scientists found that about 5% of patients had a rearrangement of a gene called MEF2D. Patients with this genetic change tended to be older than most ALL patients. They were also less likely to survive.

The scientists also figured out how the chromosome rearrangement helped fuel the leukemia.

The insight led them to the drug panobinostat. In the lab, the drug halted growth of human tumor cells with the MEF2D rearrangement. The drug is already being used to treat other types of leukemia. This study suggested it might act in a more precise, targeted way against cells with the MEF2D change.

“Further testing is underway to see if drugs like this one, either alone or in combination with chemotherapy, offer a new approach for these patients,” said Charles Mullighan, MD, MBBS, of Pathology.

The research appeared in Nature Communications.

Read the news release.