Listen to this article New Experimental Pill Shows Promise in Eliminating Leukemia Cells in Terminal Patients
Introduction
Terminal patients diagnosed with acute myeloid leukemia (AML) who have not responded to traditional treatments may now have hope for a cure. The development of a new experimental pill called revumenib has shown promising results in eliminating cancer cells in a third of the participants in a clinical trial in the United States.
The Study and Its Results
The long-awaited clinical trial, which was published in Nature, saw revumenib completely eliminate cancer cells in every patient. Although not all patients showed complete remission, scientists remain optimistic, as the drug has the potential to pave the way for a cure for leukemia in the future.
According to Dr. Ghayas Issa, a leukemia physician at the MD Anderson Cancer Center at the University of Texas and study co-author, “We’re incredibly hopeful by these results of patients that received this drug. This was their last chance. 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.”
How Revumenib Works
Acute myeloid leukemia (AML) is a type of cancer that attacks the bone marrow and causes the uncontrolled production of defective cells. Additionally, Revumenib belongs to a new class of targeted therapy for acute leukemia that inhibits a specific protein called menin. Therefore, the drug works by reprogramming leukemia cells back into normal cells.
Leukemia cells hijack the complex machinery involving menin that causes normal blood cells to turn into cancerous ones. Revumenib shuts down the engine responsible for the transformation of normal blood cells into cancerous ones, causing leukemia cells to revert to normal cells and enter remission.
According to Issa, the experimental pill targets the most common mutation in AML, a gene called NPM1, and a less common fusion called KMT2A. Combined, these mutations are estimated to occur in about 30 to 40 per cent of people with AML.
The Impact and Side Effects
The promising results of the clinical trial have already saved 18 lives. Based on the data, the US Food and Drug Administration granted revumenib “breakthrough therapy designation” to help fast-track its development and regulatory review.
The drug is relatively safe compared to standard leukemia treatments, but it has two main side effects. The first side effect impacts the heart’s electrical system, and a medical professional can detect it using an electrocardiogram (ECG).However, reducing the dosage or halting the treatment resolved the issue in all cases, according to Issa.
The second side effect of blood cancer treatments is differentiation syndrome, which refers to a group of reactions that can be potentially life-threatening. However, recognizing it early and taking appropriate measures to shut it down can effectively manage it. Issa stated that all patients who experienced differentiation syndrome in this study had their cases successfully managed without any complications.
Future Research
The study is still in its early stages, and phase II is now underway to specifically look at the effectiveness of revumenib. According to Issa, the experimental pill is not a definitive cure, but the researchers who worked on the trial remain optimistic.
“In the future, we plan to combine this pill with standard treatments that we currently have for acute leukemias,” said Issa. “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.”
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