This week, scientists describe a novel experimental pill that may be able to help patients with difficult-to-treat leukemia.
In the phase I trial, nearly half of the patients responded to the treatment, known as revumenib, and about a third of the patients experienced complete cancer remission. More research, however, is required to confirm its efficacy.
Potential Leukemia Medicine
Syndax is the company that is developing revumenib. It’s supposed to work by inhibiting a protein called menin, which appears to be important in certain types of leukemia.
These cancers are distinguished by the presence of an NPM1 gene mutation or a KMT2A gene rearrangement. These abnormalities are common in both pediatric and adult leukemia cases and are frequently difficult to treat. According to the scientists’ research, the five-year survival rate for KMT2A-related leukemia is less than 25%.
The results of the first human trial of revumenib were published in the journal Nature on Thursday. The trial included 68 patients with relapsed or refractory acute leukemia who had previously failed to respond to treatment. The majority of these patients had cancer that was caused by NPM1/KMT2A.
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Experimental Treatment
Phase I trials are intended to assess an experimental treatment’s safety and optimal dose. 53% of these eligible patients responded to revumenib, with approximately 30% (18 patients) achieving complete remission.
The researchers discovered that some patients’ cancers adapted to the treatment and developed resistance to it in a second study that looked at trial data and was published this week in Nature.
It’s good because it shows that these cancers truly rely on menin to grow and that drugs capable of disrupting how the protein is used by these cancers can kill them. This could include treating patients at an earlier stage of cancer or combining revumenib with other drugs that attack these cancers from a different perspective.
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