An experimental drug achieves complete remission of acute leukemia in 18 patients

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

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.

Of the 60 patients, 53% had some degree of remission and 30% (18 patients) had complete remission.

During the clinical trial, carried out between 2019 and 2022, of the 60 patients, 53% had some degree of remission and 30%, that is, 18 patients, had complete remission or complete remission with partial hematological recovery, indicates the study.

girl inhibitors

Of these 18 patients with complete remission, 78% had undetectable measurable residual disease after nearly two months of remission, “demonstrating the potential of girl inhibitor therapies for acute leukemia,” the researchers wrote.

“These response rates, especially the residual disease clearance rates, are the highest we’ve seen with any monotherapy used for these resistant leukemia subsets,” said Issa.

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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 treatment escape routes provides valuable information that will be needed to improve future outcomes, say the authors.

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

survival rates

Acute leukemia is usually characterized by mutation of the nucleophosmin 1 (NPM1) gene or mixed-lineage leukemia gene 1 (KMT2Ar) rearrangement, both of which have been shown to contribute to cancer progression.

Overall survival rates are low, and there are currently no approved treatments that specifically target these genetic changes.

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.


Ghayas Issa et al. “The revumenib girl inhibitor in leukemia with KMT2A rearrangement or NPM1 mutation”. Nature (March 2023).

Scott Armstrong and others. “MEN1 mutations mediate clinical resistance to Menin inhibition”. Nature (March 2023).

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