Patients with terminal leukemia who were not responding to treatment now have hope for a cure thanks to revumenib, a new experimental medication.

In a long-awaited clinical trial in the United States, this medicine fully eradicated cancer in one-third of individuals.

Although not all patients achieved complete remission, doctors remain optimistic that the drug could one day lead to a cure for leukemia.

“We’re incredibly hopeful by these results of patients that received this drug. This was their last chance,” said study co-author Dr. Ghayas Issa, a leukemia physician at the University of Texas MD Anderson Cancer Center.

“They have progressed on multiple lines of therapy and a fraction of them, about half, had disappearance of their leukemia cells from their bone marrow,” Dr. Issa told Euronews Next.

How does the pill work?

Acute myeloid leukemia (AML) is a kind of cancer that targets the bone marrow, where blood cells are created and causes uncontrolled cell production.

Revumenib is a new type of targeted medication for acute leukemia that works by inhibiting a protein called menin. The medication works by converting leukemia cells to normal cells.

Menin is a part of the intricate machinery that is hijacked by leukemia cells and causes normal blood cells to become malignant.

Issa reported that by taking revumenib, the engine is shut off and leukemia cells are converted back into normal cells, resulting in remission.

This solution has already saved 18 lives as part of a clinical experiment, the results of which were recently published in Nature.

Based on the results of this trial, the US Food and Drug Administration gave revumenib “breakthrough therapy designation” in December 2022 to help expedite its development and regulatory assessment.

A promising cure, but it’s not for everyone

Issa stated that this is unquestionably a breakthrough and the result of years of scientific research.  According to him, many groups had to work hard in the lab to figure out what was producing this leukemia.

That said, he made sure to clarify that the medicine does not work for all people. It is just for a subgroup of leukemias that have missing or mislabeled genes or a chromosomal fusion.

The experimental tablet targets the most prevalent mutation in AML, a gene called NPM1, as well as a less common fusion called KMT2A. These mutations are predicted to be present in 30 to 40 percent of persons with acute myeloid leukemia.

Nine American hospitals enrolled 68 phase I patients. They all had leukemia that returned after earlier therapies or that never responded to standard chemotherapy.

Algimante Daugeliate, a 23-year-old Lithuanian architect battling leukemia, was among them. Her sister gave her two bone marrow transplants, but other therapies failed. Her doctors explored palliative care.

Two years after starting revumenib, Daugeliate recovered. After graduating college and working at a Copenhagen architecture studio, she has returned to everyday life.

Great impact, limited side effects

Although this medicine is quite safe when compared to typical leukemia treatments, two major side effects have been detected, according to Issa.

The first affects the electrical system of the heart and can be discovered with an electrocardiogram (ECG). However, decreasing the dosage or discontinuing treatment corrected the problem in all cases, Issa said.

The second side effect is known as differentiation syndrome, which is a series of potentially fatal reactions to blood cancer treatments. It can be effectively handled if it is recognized early and proper measures are taken to stop it. All cases of differentiation syndrome in this study, according to Issa, were successfully managed with no problems for the patients.

A promising study, but further investigation is needed

The research is still in its early phases, and the findings are tentative. Phase I studies, such as this one, are designed to determine whether a medicine is safe and to determine the greatest dose that may be administered without generating serious side effects.

A phase II study examining the efficacy of revumenib has also been conducted.

Twelve trial participants who reacted to the medication received a stem cell or bone marrow transplant. Such transplants require patients to have no or very low quantities of malignancy in their blood, which revumenib helped them achieve.

While the experimental drug is not a cure-all, the researchers involved in the trial are optimistic.

“In the future, we plan to combine this pill with standard treatments that we have currently for acute leukemias,” Issa said.

“That is the most likely strategy to get us to cures where patients don’t have to see leukemia doctors after that and don’t need treatments for leukemia.”