New Experimental Drug May Cure Leukemia

A clinical trial conducted in the United States revealed that a new experimental drug is showing great promise in the fight against leukemia since it eradicated the disease in 18 terminally ill patients.

“We’re incredibly hopeful by these results of patients that received this drug. This was their last chance,” explains study co-author Dr. Ghayas Issa, a leukemia physician at the MD Anderson Cancer Center at the University of Texas. “They have progressed on multiple lines of therapy and a fraction of them, about half, had disappearance of their leukemia cells from their bone marrow.”

The experimental pill called revumenib is part of a new class of targeted therapy that inhibits the specific protein menin partly responsible for turning normal blood cells into cancerous ones. The drug reprograms leukemia cells back into normal cells. Out of the 54 patients taking part in the clinical trial, 18 were cured. According to the study, 53% of patients responded to revumenib and 30% had a complete remission, with no cancer detectable in their blood. The experimental drug works on the gene called NPM1 and a fusion called KMT2A, two of the most common mutation in acute myeloid leukemia. A phase II study is currently underway, looking at the effectiveness of revumenib. “In the future, we plan to combine this pill with standard treatments that we have currently for acute leukemias.”