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This disease, a variant of cancer, can affect adults and children.
18/3/2023
Leukemia is a variant disease of cancer, according to the American Cancer Society. “Cancer is in the primitive blood-producing cells. Most often, leukemia is a cancer of the white blood cells, but some leukemias start in other types of blood cells.”
It is important to note that among the characteristics of this disease are multiple variants, depending on the type of blood cell that is affected by the condition, as well as its severity.
Also, apparently it might be cured, but only if it meets a specific characteristic. This was discovered by a team of researchers from the MD Anderson Cancer Center of the University of Texas (United States).
The scientists discovered that this might be cured in its advanced stage, following achieving it in 18 patients thanks to the administration of the experimental drug revumenibformerly known as Sndx-5613, according to the results of the phase I study ‘Augment-101’, published in the journal Nature.
In time, it was revealed that the overall response rate among 60 patients was 53%, and the rate of complete remission or complete remission with partial haematological recovery was 30%. In addition, 78% of those affected achieved clearance of measurable residual disease.
“These results suggest that revumenib may be an effective targeted oral therapy for patients with acute leukemia caused by genetic alterations.. These response rates, especially the residual disease clearance rates, are the highest we have seen with any monotherapy used for these resistant leukemia subgroups,” explained study leader Ghayas Issa.
The types of diseases that were studied included acute myeloid leukemia (82%), acute lymphocytic leukemia (16%), and acute leukemia of mixed phenotype (2%). Among the included patients, 67.6% had KMT2A rearrangements, 20.6% had NPM1 mutations, and 11.8% had other genotypes.
Patients received intensive pretreatment with a median of four prior lines of therapy and 46% had a prior allogeneic stem cell transplant. The response duration measure was 9.1 months and the median overall survival was 7 months. Thus, 12 patients underwent allogeneic stem cell transplantation following response to revumenib.
This is the first evidence showing the safety and clinical activity of menin inhibition, induced by said treatment, in acute leukemia.and the data demonstrate the potential to target scaffold proteins that have been shown to be vulnerable points in specific cancers.
The researcher explained that targeting menin disrupts the gene transcription machinery and changes gene expression in cancer cells from a leukemia pattern to a normal pattern, ultimately leading to remission.
“Responses in this trial show that menin inhibitors may be a promising treatment option that is well tolerated by patients and might be the most recent addition to successful targeted therapies for acute leukemia. I look forward to additional data from this and future trials to inform the potential opportunity to offer this specific treatment to more patients.”
Enrollment for the Phase II trial cohort is ongoing. Future trials of revumenib will test combinations with other agents in various settings, including newly diagnosed leukemias, relapsed or refractory disease, and maintenance therapy, for leukemias with KMT2A rearrangement or NPM1 mutation, and other leukemias amenable to menin inhibition.
*With information from Europa Press.
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