A survey by the MD Anderson Cancer Center at the University of Texas in the United States suggested a possible strategy for treating patients acute leukaemias with mutations and for which no specific therapy has yet been approved for use. With an experimental treatment with the drug revumenib in a clinical phase 1 study, the scientists achieved complete remission of the disease in a total of 30% of the volunteers 18 diseasefree patients. The next phase of the study, published in Natureis to review the impact in other scenarios, such as in newly diagnosed leukemia and in maintenance therapies.
In the study, researchers tested the drug on participants advanced acute leukemias with KMT2A rearrangements or Mutant NPM1. Although they do not represent the majority of patients with this disease, these types are challenging and important for the development of new treatments.
“Acute leukemias with KMT2A rearrangements are difficult to treat, and NPM1 mutations are the most common genetic alteration in acute myeloid leukemia. These subgroups have not approved any specifically targeted therapies,” study leader Ghayas Issa said in a statement from the university.
The drug manages to inhibit a protein called “menin,” or MEN1, which plays a crucial role in the expression of genes that promote leukemia. This protein has been the subject of study because preclinical studies indicated that interfering with its interaction with mutations can be an effective measure to eliminate cancer cells.
According to the researcher, with the inhibition of the protein, a process of genetic modification of the tumor cells takes place, which go into a normal pattern. In this way, the patient has remission.
The scientists succeeded in doing this in the study 53% overall response rate, among the 60 participants, who had an average age of 43 years, but had babies as young as 10 months among the volunteers. A complete remission rate was 30%. “These response rates, particularly residual disease cure rates, are the highest we have seen for any monotherapy used for these resistant leukemia subgroups,” Issa said.
The main side effects of the treatment were diarrhea (3%), fatigue (3%) and anemia (3%). No volunteer dropped out of therapy because of them.
“These inhibitors could be a promising treatment option that is well tolerated by patients and could be the latest addition to successful targeted therapies for acute leukemia,” he said.
Continued after the ad
Abril Comunicações SA, CNPJ 44.597.052/000162 All rights reserved.
Brazil is changing. The whole time.
Follow him on VEJA and also have access to the digital content of all other Abril titles*
Quality and reliable information just a click away.
* Unlimited digital access to the websites and editions of the digital magazines in the apps: Veja, Veja SP, Veja Rio, Veja Saúde, Claudia, Superinteressante, Quatro Rodas, Você SA and Você RH. * Annual payment of BRL 96, equivalent to BRL 2 per week.