Researchers have investigated whether the drug revumenib works in children and adults whose leukemia has returned or did not respond well to standard treatment. Revumenib is a menin (protein) inhibitor. The drug switches off the genetic KMT2A abnormality that forms a protein complex together with menin. Because the drug blocks the formation of this protein complex, some leukemia cells disappear.
The study shows that nearly 1 in 4 children (23%) went into remission after treatment with revumenib. This means that no leukemia cells were found in their blood or bone marrow. This is a clear improvement over previous treatments, when 1 in 10 children went into remission.
A total of 94 people from different countries participated in this study, including 23 children. Five children were treated at the Princess Máxima Center. The Máxima was the only European hospital to participate in this study.
Children responded just as well to the treatment as adults. They were given an adjusted dose of the medication. Nearly 65% of all participants showed a positive response to the medication. In these cases, the leukemia disappeared completely or partially.
New type of medicine
Revumenib belongs to a new group of medicines: menin inhibitors. These medicines block a protein (menin). In children and adults with a KMT2A abnormality, this protein causes leukemia cells to grow. By inhibiting this protein, the leukemia cells can disappear.
The drug also has advantages in terms of quality of life. It is administered as a drink or pill and does not usually cause nausea. In the future, it may even be administered on an outpatient basis, so your child will not have to stay in the hospital.
Next step: new study
The Princess Máxima Center will soon start a new study with another drug from this group: ziftomenib. This drug is also a menin inhibitor. Researchers want to determine the safe dosage for children with acute leukemia and a specific genetic abnormality. The study is being led by Prof. Michel Zwaan's research group and coordinated by the Máxima's Trial and Data Center, together with the American Leukemia Lymphoma Society.
The ultimate goal is to add these drugs to the standard treatment. We hope that this will enable more children with an aggressive form of leukemia to recover in the future.
Would you like to read the scientific publication? Please look here: Issa GC, et al. Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101). J Clin Oncol. 2025 Jan;43(1):75-84.