An anti-cancer drug could potentially cure acute myeloid leukemia

According to the National Cancer Institute, over 11,000 people in the U.S. could lose their life this year because of acute myeloid leukemia (AML), a type of blood cancer resulting from the uncontrolled production of immature white blood cells in the bone marrow. 

Although AML can be treated using chemotherapy, there is still no guaranteed cure for this deadly disease. Between 2019 and 2022, a team of scientists conducted phase one clinical trials of an anti-cancer drug called revumenib on patients living with AML. 

The results from the trial were recently published in two studies; one highlights the effectiveness of revumenib in treating AML, whereas the other sheds light on the resistance leukemia cells developed against the drug, proving its potential as a possible AML treatment approach. 

What causes AML?     

The study authors explain that AML is triggered by mutations in two genes named KMT2Ar and NPM1, and a protein called menin causes the progression of these cancer-causing mutations. Menin is encoded by the MEN1 gene, and it is actually responsible for preventing uncontrolled cell division and growth in the human body. 

However, instead of keeping a check on cell division, menin has been found to be facilitating the mutations that lead to out-of-control cell multiplication in AML patients. This is where revumenib comes into play, this drug has the power to selectively inhibit menin. 

President of Dana-Farber Cancer Institute and one of the study authors, Scott Armstrong, told IE, “Revuminib blocks a critical protein menin that has been previously shown to be necessary for the mutant proteins to keep AML cells alive and dividing.” 

How can revumenib help?

During the first study, the researchers tested revumenib on 60 AML patients. They noted a 53 percent response rate, meaning 53 percent of patients showed some AML remission. Moreover, 18 out of 60 patients experienced complete remission, and in 14 of them, the cancer cell count dropped to undetectable levels just after two months of receiving the treatment.

What’s more exciting is that the authors of the second study found new mutations in the MEN1 genes of some patients after they took revumenib. These mutations were aimed at developing resistance against the drug.  

“In some leukemias that developed resistance to Revumenib, the MEN1 gene (encoding the Menin protein) developed a mutation that decreased the binding power or activity of Revumenib. This shows that leukemia is highly dependent on Menin function and validates this therapeutic approach,” said Armstrong.

A possible cure for AML

The effectiveness of revumenib in inhibiting menin and the resistance further showed by the cancer-driving genes against this drug proves that it has the potential to give rise to an AML cure that will benefit millions of people across the globe. 

The researchers claim that it’s even better than standard AML treatment methods because it is less complex than chemo and is still able to bring a better response rate. Ghayas Issa, one of the researchers and a professor at The University of Texas, told IE: 

“In adults with KMT2A rearranged leukemias, chances of responding to standard therapy once 3 prior attempts have been made (similar to the study population) is ~ nine percent, whereas we show in this study that chances of responses to revumenib with complete remission or complete remission with a partial hematologic recovery of 30 percent.”

The researchers are now busy conducting phase two trials. Hopefully, soon we’ll see the rise of a new and better AML treatment method.

The first and second studies are published in the journal Nature.