From the article: One of the papers presents results of a clinical trial in which approximately 40% of patients with acute leukemia subtypes had a complete response – a disappearance of all signs of cancer – to treatment with the drug revumenib. The other paper uncovers a molecular countermove by which leukemia cells come to sidestep the drug and reassert their growth.

The papers point to the promise of the targeted approach to acute leukemia treatment exemplified by revumenib and to the potential to extend its benefits with drugs that trip up the resistance mechanism, researchers say.

"The two genetic subtypes of acute leukemia involved in this research account for approximately 40% of all cases of acute myeloid leukemia (AML) in children and adults," says Scott Armstrong, MD, PhD, president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and co-senior author of the paper on revumenib resistance. "They're driven by a rearrangement of the MLL1 gene or a mutation in the NPM1 gene. Both types depend on a protein called menin to sustain their growth."

The first of the new Nature studies reports on a phase I/II clinical trial of the drug revumenib, which targets menin, in 68 patients with acute leukemia that wasn't responding other therapies. The trial, dubbed the AUGMENT-101 study, found that of 60 patients who could be evaluated, 53% responded to the drug and 30% had a complete response.

"For patients with acute leukemia who have undergone several previous treatments, this is a very encouraging result," Armstrong says. "However, after the second cycle of treatment, some patients did develop resistance to revumenib."