Revumenib, an experimental pill, targets gene mutations NPM1 and KMT2A, which occur in about 30-40% of people with acute myeloid leukaemia. The promising phase 1 clinical trial results have shown that revumenib can provide a full recovery for terminal leukaemia patients who were unresponsive to other treatments. Although the drug is generally safe, there are two main side effects identified. A phase II study to evaluate the drug’s effectiveness is now underway. Dr Ghayas Issa, a leukaemia physician at the University of Texas, plans to combine revumenib with standard treatments to achieve a cure for leukaemia where patients will not require further treatment.
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New Experimental Pill Shows Hope for Leukaemia Patients
Revumenib, an experimental drug, has provided a new ray of hope for terminal leukaemia patients who were unresponsive to treatment.
A clinical trial conducted in the United States showed that the drug has eliminated cancer in a third of the participants. While not all patients experienced complete remission, the results are promising, indicating that revumenib might lead to a cure for leukaemia in the future.
How Revumenib Works
Revumenib, a new class of targeted therapy, inhibits menin, a specific protein involved in the complex machinery that causes normal blood cells to turn into cancerous ones. The drug works by reprogramming leukaemia cells back into normal cells, leading to remission.
The clinical trial’s preliminary results revealed that 53% of patients responded to the drug, while 30% experienced complete remission with no detectable cancer in their blood.
Promising Results
The drug’s success has been hailed as a significant breakthrough by Dr Ghayas Issa, a leukaemia physician at the University of Texas. The US Food and Drug Administration granted revumenib “breakthrough therapy designation” in December 2022, based on the data from the clinical trial.
However, Issa explained that revumenib is not effective for all leukaemia patients. It is tailored for a specific subset of leukaemia patients who have missing or mislabeled genes or a chromosome fusion.
Revumenib has already saved 18 lives, and the promising results of the clinical trial were published this month in Nature.
Experimental Pill Targets Most Common Leukaemia Mutations
Revumenib, an experimental pill, targets the most common mutation in acute myeloid leukaemia, a gene called NPM1, and a less common fusion called KMT2A. Together, these mutations occur in approximately 30 to 40% of people with acute myeloid leukaemia.
Promising Phase 1 Clinical Trial Results
The phase 1 trial involved 68 patients across nine US hospitals, all of whom had experienced a return of their leukaemia after other treatments or had never responded well to traditional chemotherapy drugs.
Among the trial participants was Algimante Daugeliate, a 23-year-old Lithuanian architect diagnosed with leukaemia. After all other treatments had failed, revumenib provided a full recovery and allowed her to resume her normal life, graduating from college and working at an architecture studio in Copenhagen.
Although the study is still in its early stages, the preliminary results are promising. Phase 1 studies like this aim to determine a drug’s safety and the highest dose that can be given without causing severe side effects.
Limited Side Effects
Revumenib is generally safe when compared to standard treatments for leukaemia. However, two main side effects have been identified. The first affects the heart’s electrical system, which can be detected with an electrocardiogram (ECG). Reducing the dosage or halting the treatment resolved the issue in all cases.
The second side effect is known as differentiation syndrome, a group of potentially life-threatening reactions to blood cancer treatments. It can be managed effectively if it is recognised early, and appropriate measures are taken to shut it down. All cases of differentiation syndrome in the study were successfully managed without any complications for the patients.
Phase II Study Underway
A phase II study specifically looking at the effectiveness of revumenib is now underway. Twelve patients who responded to the drug in the trial went on to receive a stem cell or bone marrow transplant. Such transplants require that patients have no cancer or only very low levels of cancer in their blood – and revumenib helped them get there.
While revumenib is not a definitive cure, the researchers who worked on the trial are optimistic about the future. Dr Ghayas Issa, a leukaemia physician at the University of Texas, plans to combine this pill with standard treatments currently available for acute leukaemias.
Revumenib is a promising development in the field of leukaemia treatment and may provide hope for those who do not respond to other treatments.
A Promising Strategy for Leukaemia Cures
According to Dr Ghayas Issa, a leukaemia physician at the University of Texas, combining revumenib with standard treatments is the most likely strategy to achieve a cure for leukaemia where patients will not require further treatment.
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