Breakthrough experimental drug revumenib has shown significant promise for leukemia treatment in early clinical trials. A third of the patients with acute myeloid leukemia (AML) found themselves in complete remission during the long-awaited trial.

The results were described as “very promising” by blood cancer expert Dr. Scott Armstrong, who works at the Dana-Farber Cancer Institute in Boston.

Should subsequent trials be successful, it is believed the drug could get Food and Drug Administration (FDA) approval before the end of the year.

What Is AML?

Acute myeloid leukemia (AML), a rather aggressive disease, is a blood cancer that targets white blood cells, leaving a person unable to fight infection. Because these cells are rather diverse and can mutate rapidly, even novel cancer treatments have little effect.

According to statistics, around 474,000 people around the world were diagnosed with AML. In the US alone, 59,000 people are diagnosed each year and 40% of them end up dying.

AML is most common among adults over 45 but can happen to anyone at any age.

At the moment, chemotherapy is the main course of treatment for this type of cancer, and radiotherapy can be used if the cancer has spread significantly throughout the body. Surgery is rarely used.

Nature, a British-based scientific journal, says that 40% of US leukemia cases are caused by a mutation in the NPM1 and KMT2A genes.

“In leukemia with these mutations, a protein called menin binds to genes that trigger cancerous cells to keep growing and dividing.”

Dr. Eytan M. Stein, states that “The outcomes of patients with acute leukemia with these genetic abnormalities, especially when relapsed or refractory to treatment, [are] very poor — median overall survival is measured in months. New therapies are desperately needed to give these patients a meaningful therapeutic option.”

Revumenib, it appears, may be the long-awaited miracle that doctors have been searching for. It is a menin inhibitor, meaning that it prevents menin from attaching to the genes, thus breaking the cancerous cycle.

The Revumenib Trial

A total of 68 patients, most of which had the aforementioned gene mutations, participated in this clinical trial. Over about a course of a year, on average, all participants were instructed to take revumenib in pill form twice a day, approximately 12 hours apart.

In the end, around 53% of the patients showed to have partial remission, while 30% had complete remission. Scientists working on the experiment said that it took two months on average to reach complete remission.

Unfortunately, almost all of the participants experienced severe side effects, and seven people had to withdraw from the study completely.

Armstrong also reported that “after the second cycle of treatment some patients did develop resistance to revumenib.” 

Considering that the first part of any clinical trial is to “test the safety and optimal dose of an experimental treatment,” revumenib will likely move on to the next phase. However, it will be necessary to first examine current patients and address the side effects first.

According to Medical News Today, there have been several trials that show menin inhibitors can help in leukemia treatment. And revumenib may be the most promising one yet.