CPHI China 2023 News: New small molecule drugs promote leukemia remission, study says

A team of scientists at the University of Florida Cancer Research Center said that Revumenib, a new drug developed by the team, can reduce the abnormal proliferation of white blood cells to relieve symptoms in patients. CPHI learned that the team's Phase I/IIa clinical trial for the safety and effectiveness of Revumenib combined with chemotherapy in the treatment of acute myeloid leukemia began in January 2020, with a total of 30 volunteers.

Revumenib is a small molecule compound drug whose mechanism of action is to alleviate the course of disease in leukemia patients by inhibiting the interaction between Menin and MLL1.

MEN1 is a tumor suppressor gene that controls cell proliferation and DNA repair. If the MEN1 gene is mutated, the protein is inactivated and cancer cells grow unchecked.

The team said that during the experiment, there were no major side effects in patients with acute myeloid leukemia who received Revumenib combined with chemotherapy, and most patients had certain relief of symptoms, and the inhibition of cancer cell proliferation was good.

According to a study published in Nature, 68 patients with acute leukemia who did not respond to other therapies participated in the Phase I/II clinical trial of revumenib, and 60 groups of effective data were obtained, of which 53% responded to the drug and 30% had a good response.

However, after the second treatment cycle, some patients developed resistance to revumenib.

The researchers analyzed the causes of drug resistance in follow-up studies and found that bone marrow samples from leukemia patients who had developed resistance to revumenib had mutations in MEN1 in many of them, causing the receptor to fail to bind to revumenib and the growth of cancer cells to be uninhibited.

This also demonstrates that MEN1 gene mutation can be an important reference for assessing the risk of recurrence in patients with both inherited subtypes of acute myeloid leukemia (AML).

"This is very good news for patients with acute leukemia who have been treated several times," Armstrong said.

Dana-Farber's Dr. Richard Stone, a participant in the study, said he was positive about the drug's efficacy in treating acute leukemia and hoped to evaluate its safety and effectiveness in further trials.