A new experimental pill called revumenib has brought hope to terminal leukemia patients who had not responded to previous treatments, as it has eliminated cancer in a third of the participants in a clinical trial conducted in the United States.
While not all patients achieved complete remission, the results are encouraging, suggesting that the pill may offer a potential cure for leukemia in the future.
“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 MD Anderson Cancer Center at the University of Texas.
“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,” he told Euro news Next.
Acute myeloid leukemia (AML) is a type of cancer that attacks the bone marrow, causing the production of defective cells. Revumenib is a targeted therapy for acute leukemia that inhibits a protein called Menin, which is involved in the machinery that causes normal blood cells to turn into cancerous ones.
By reprogramming leukemia cells back into normal cells, the drug turns off the engine that gets hijacked by leukemia cells, leading to remission.
As part of the clinical trial, 53% of patients responded to revumenib, with 30% achieving complete remission and no detectable cancer in their blood. The trial’s promising results were published in Nature, and based on the data, the US Food and Drug Administration granted revumenib “breakthrough therapy designation” in December 2022 to help expedite its development and regulatory review. So far, the drug has already saved 18 lives.
“This is definitely a breakthrough and it’s a result of years of science. A lot of groups had worked hard in the lab to understand what is causing these leukemias,” Issa said.
However, he explained that the drug does not work for all patients. It is for a specific subset of leukemias that generally have missing or mislabeled genes or a chromosome fusion.
Two main side effects have been identified for the new drug revumenib, although it is considered relatively safe when compared to standard treatments for leukemia, according to Issa.
The first side effect affects the heart’s electrical system and can be detected with an electrocardiogram. However, reducing the dosage or halting the treatment resolved the issue in all cases, Issa noted.
The second side effect, known as differentiation syndrome, can be life-threatening, but all cases in the study were effectively managed without complications for the patients if it was recognized early and appropriate measures were taken to shut it down.
The study is in its early stages, and the results are still preliminary. Phase I studies like this one aim to determine the safety of a drug and find the highest dose that can be administered without causing severe side effects. A phase II study focusing on the effectiveness of revumenib is currently underway. In the trial, 12 patients who responded to the drug received a stem cell or bone marrow transplant, which requires patients to have no or very low levels of cancer in their blood.
While revumenib is not a definitive cure, the researchers are optimistic. The drug has demonstrated promising results, helping patients achieve remission and paving the way for future treatments.
“In the future, we plan to combine this pill with standard treatments that we have currently for acute leukemias,” said Issa.
“That is the most likely strategy to get us to cure where patients don’t have to see leukemia doctors after that and don’t need treatments for leukemia”.