Inside something study outside this week the researchers describe a new experimental pill can help patients with leukemia that is difficult to treat. Almost half of the patients in the phase I trial responded to revumenib treatment, while about a third experienced complete remission of their cancer. However, more research is needed to confirm its effectiveness.
Revumenib is developed by the Syndax company. It is thought to work by blocking a protein called menin, which appears to play an important role in certain forms of leukemia. These cancers are characterized by an NPM1 gene mutation or a rearrangement of another gene called KMT2A. These abnormalities are common in both childhood and adult cases of leukemia and are often very difficult to treat. By research For example, the five-year survival rate cited by researchers for KMT2A-related leukemia is less than 25%.
On Thursday, these researchers published the results of the first human trial of revumenib in the journal Nature. The study included 68 patients with relapsed or refractory acute leukemia who had not responded to previous treatments. The majority of these patients had NPM1/KMT2A-related cancer.
Phase I trials are designed to test the safety and optimal dose of the experimental treatment. But to understand its potential power, the team also looked at the results of patients with NPM1/KMT2A-related leukemia (60 in total). Of these eligible patients, 53% showed some response to revumenib and approximately 30% (18 patients) achieved complete remission. Although almost all patients experienced side effects, including potentially serious ones such as irregular heartbeat, none had to stop treatment as a result.
“For patients with acute leukemia who have received multiple prior therapies, this is a very encouraging result,” said study author Scott Armstrong, MD, director of Dana-Farber and Boston Children’s Cancer and Blood Disorders Center. opinion Published by Dana-Farber.
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Phase I trials are just the beginning of clinical development, and many drugs that show encouraging results early on fail to live up to that promise in larger trials. And while revumenib proves largely effective against these cancers, it may not be long-lasting for some. Inside something another study Also reviewing research data published this week in the journal Nature, researchers found that some patients’ cancers adapted to the treatment and developed resistance to it.
These findings are both good news and bad news, the authors say. It’s good, because it shows that these cancers actually rely on menin to promote their growth and likewise that drugs that can interfere with the use of the protein by these cancers can sterilize them. At the same time, it likely also means that other strategies are needed to delay or prevent resistance to these drugs. This could include treating people with earlier-stage cancer or combining revumenib with other drugs that attack these cancers from a different angle.
An expansion of the study is already underway, with full Phase I/II results expected later this year. If things continue well, the company is done said it could begin seeking FDA approval as early as the end of 2023. If approved, revumenib would be the first menin inhibitor to be used in the treatment of cancer.