An experimental drug manages to remit acute leukemia in 18 patients

An experimental drug for advanced or resistant acute myeloid leukemia has, in a small clinical trial with 60 people, achieved some degree of remission in 53% of patients and complete remission in 30%. There are 18 people who have seen their disease, blood cancer and bone marrow of this most common type in adults, remit. Two studies, published in ‘Nature’, also show that possible signs of resistance to treatment have been detected.



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The 60 people who participated in the clinical phase 1 trial received the experimental drug by mouth revimenib, which, according to the journal, “has shown anticancer effects and possible indications of resistance.”

The first study, led by oncologist Ghayas Issa, from the University of Texas (USA), showed that the inhibition of a protein called menin thanks to the use of this drug “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 therapy for patients with acute leukemia caused by these genetic alterations,” he said in a statement.

During the clinical trial, conducted between 2019 and 2022, more than half of the patients (53%) had some degree of disease remission and 30% had a complete remission, the study says. Of these 18 patients, 78% had undetectable measurable residual disease after almost two months of remission, “demonstrating the potential of menin inhibitor therapies for acute leukemia,” the researchers write. “These response rates, especially the residual disease clearance rates, are the highest we have seen with any monotherapy used for these resistant leukemia subsets,” Issa said.

The second of the studies, led by researcher Scott Armstrong, from the Dana Farber Cancer Institute (USA), delved into the appearance of selective resistance to menin inhibition. The team identified specific mutations in the MEN1 gene, which encodes menin, which can lead to resistance to revumenib treatment through disruption of the drug binding site. These mutations were detected in several patients who initially responded to treatment but did not maintain the clinical response. Identifying these escape routes from treatment provides valuable information that will be needed to improve patient outcomes in the future, according to the publication.

Acute leukemia is usually characterized by either the nucleophosmin 1 (NPM1) gene mutation or the mixed lineage leukemia 1 (KMT2Ar) gene 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 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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