Breaking: Experimental new drug achieves ´encouraging´ results for complete remission in leukemia patients

An investigational drug for advanced or resistant acute myeloid leukemia achieved, in a small clinical trial of 60 people, some degree of remission in 53% of patients and complete remission in 30% (18 people), although possible signs of remission were also detected.

Two studies published this week in Nature present the results of a phase 1 clinical trial involving 60 people who were treated with the experimental oral drug revumenib, which “revealed anticancer effects and possible signs of resistance,” the journal notes.

The first study, led by Ghayas Issa of the University of Texas, showed that inhibition of a protein called girl using revumenib “produced encouraging responses” in advanced acute leukemias with mutant KMT2A or NPM1 rearrangements.

“I am encouraged by these results, which suggest that revumenib may be an effective oral therapy for patients with acute leukemia caused by these genetic alterations,” Issa said in a university statement.

The second of the studies, led by Scott Armstrong, from the Dana Farber Cancer Institute (USA), investigated the appearance of selective resistance to the girl’s inhibition.

The team identified specific mutations in the MEN1 gene (which codes for girl) that could lead to resistance to revumenib treatment through disruption of the drug’s binding site.

These mutations were detected in several patients who initially responded to revumenib treatment but did not maintain clinical response.

Identifying these treatment escape routes provides valuable information that will be needed to improve patient outcomes in the future, according to the publication.

Previous preclinical studies have shown that the girl protein facilitates the progression of KMT2Ar or NPM1 mutated acute leukemia, indicating that inhibition of this protein can reverse cancer progression in this subset of leukemias.

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