An experimental drug achieves cancer remission in 18 people with acute leukemia

An experimental drug for advanced or resistant acute myeloid leukemia has 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 resistance to treatment have also been detected.

Two studies published by ‘Nature’ this Wednesday present the results of a trial in clinical phase 1 in which 60 people participated who were treated with the experimental drug by mouth revumenib, which “has revealed anticancer effects and possible indications of resistance,” the journal notes.

The first study, led by Ghayas Issa of the University of Texas, showed that inhibition of a protein called menin through the use of revumenib, “produced encouraging responses” in advanced acute leukemias with KMT2A rearrangements or mutant NPM1. “I am encouraged by these results, which suggest that revumenib may be an effective oral targeted therapy for patients with acute leukemia caused by these genetic alterations,” Issa said in a university statement.

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, showed complete remission or complete remission with partial haematological recovery, the study indicates. Of those 18 patients with complete remission, 78% had undetectable measurable residual disease after almost two months of remission, which “demonstrates the potential of menin inhibitor treatments for acute leukemia“, the researchers write.

The conclusions of the second study

The second of the studies, led by Scott Armstrong of the Dana Farber Cancer Institute (USA), delved into the emergence of selective resistance to menin inhibition. The team identified specific mutations in the MEN1 gene (encoding menin), which can lead to resistance to revumenib treatment through disruption of the drug binding site.

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These mutations were detected in several patients who initially responded to revumenib treatment but who they did not maintain the clinical response. Acute leukemia is usually characterized by nucleophosmin 1 (NPM1) gene mutation or mixed lineage leukemia 1 (KMT2Ar) gene rearrangement, and it has been shown that both contribute to cancer progression.

Overall survival rates are low and there are currently no approved treatments. that specifically target these genetic alterations. Previous preclinical studies had shown that menin protein facilitates the progression of acute leukemia with KMT2Ar or NPM1 mutation, indicating that inhibition of this protein could reverse cancer progression in this subset of leukemias.

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