A new experimental pill called revumeni has given hope of a cure to terminally ill leukemia patients who did not respond to treatment. In a clinical trial conducted in the United States, the drug has completely eliminated the cancer in a third of the participants. Although not all patients showed a complete remission, the scientists remain hopeful as the results indicate that the pill could pave the way for a leukemia cure in the future.
Revumenib is a new class of targeted therapy for acute leukemia that inhibits a specific protein called menin. The drug works by reprogramming the leukemia cells so that they become normal again. The experimental pill targets the most common mutation in acute myeloid leukemia, a gene called NPM1, and to a less common fusion known as KMT2A.
The promising results of the trial were published this month in the scientific journal Nature. Based on the data from this trial, in December 2022, the US Food and Drug Administration (FDA) gave revumenib the designation of innovative therapy to help speed up their development and regulation.
Although this drug is fairly safe compared to standard leukemia treatments, there have been two main side effects: affecting the electrical system of the heart and differentiation syndrome – a group of life-threatening reactions to blood cancer treatments. However, all the differentiation syndrome cases in this study were treated successfully and without any complications for the patients.
The project is still in an initial phase, and its results are still preliminary. A phase II study specifically looking at the efficacy of revumenib is currently underway. The researchers who worked on the trial are optimistic about the future and plan to combine this pill with the standard treatments now available for acute leukemias, which is the strategy most likely to lead to a cure where patients later don’t have to see leukemia doctors and don’t need further leukemia treatment.