Researchers cured advanced leukemia in 18 patients thanks to the administration of the experimental drug revumenibaccording to the results of the phase I studio ‘AUGMENT-101′, published in the magazine nature.

Specifically, the general response rate among 60 patients was 53%, and the complete remission rate or complete remission with partial hematologic recovery was 30%. In addition, 78% of those affected achieved the elimination of measurable residual disease.

“These results suggest that revumenib can be an effective targeted oral therapy for patients with acute leukemia caused by genetic alterations“, says Ghayas Issa, leader of the study, carried out at the MD Anderson Cancer Center of the University of Texas (United States)

The specialist added that “these response rates, especially the residual disease elimination rates, are the highest we have seen with any monotherapy used for these resistant subgroups of leukemia”.

This is the first evidence that demonstrates the safety and clinical activity of the inhibition of the girl, induced by different treatments, in acute leukemia, and the data demonstrate the potential to address the andamium proteins that have been shown to be vulnerable points in specific cancers, details Europe Press.

According to Issa, addressing the girl interrupts the gene transcription machinery and changes gene expression in cancer cells from a leukemia pattern to a normal pattern, which finally leads to remission.

The trial enrolled 68 patients with a median age of 43 years, including children up to 10 months of age.

The types of illnesses included:

  • Acute myeloid leukemia (82%)
  • Acute lymphocytic leukemia (16%)
  • Acute leukemia of mixed phenotype (2%)

Among the patients included, 67.6% had rearrangements of KMT2A20.6% had mutations in NPM1 and 11.8% had other genotypes.

Patients received intensive pretreatment with an average of four previous therapy lines and the 46% had an allogeneic transplantation of mother prior cells.

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The median duration of the response was 9.1 months and the median overall survival was seven months. Two patients have undergone a mother cell allotransplantation after responding to revumenib, a drug formerly known as SNDX-5613.

“The responses in this trial show that the girl’s inhibiters can be a promising treatment option that patients tolerate well and could be the most recent incorporation into successful targeted therapies for acute leukemia. I look forward to further data from this and future trials to inform the potential opportunity to offer this specific treatment to more patients.”, emphasized the expert.

Next phase II trials with revumenib are expected to test other types of leukemia. (I)

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