In a from studying this week scientists describe a new experimental pill that patients with a difficult to treat form of leukemia. Nearly half of the patients in the phase I trial responded to the treatment, called revumenib, while about a third experienced complete remission of their cancer. However, more research will be needed to confirm its effectiveness.
Revumenib is being developed by the company Syndax. It is intended to work by inhibiting 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 pediatric and adult cases of leukemia and are often very difficult to treat. According to research as cited by the scientists, the five-year survival rate for KMT2A-related leukemia is less than 25%.
On Thursday, these researchers published results from the first human trial of revumenib in the journal Nature. The trial enrolled 68 patients with relapsed or refractory acute leukemia who had failed to respond to previous treatments. Most of these patients had NPM1/KMT2A-related cancer.
Phase I trials are designed to test the safety and optimal dose of an experimental treatment. But to understand its potential efficacy, the team also looked at the outcomes of people with NPM1/KMT2A-related leukemia (60 in total). Of these eligible patients, 53% had some response to revumenib and approximately 30% (18 patients) were in complete remission. Although almost all patients experienced side effects, including potentially serious ones such as an irregular heartbeat, none had to stop treatment as a result.
“For patients with acute leukemia who have had several previous treatments, this is a very encouraging result,” study author Scott Armstrong, president of the Dana-Farber and Boston Children’s Cancer and Blood Disorders Center, said in a statement. rack released by Dana-Farber.
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Phase I trials are just the beginning of clinical development, and many drugs that show encouraging early-stage results fail to deliver on that promise in larger trials. And even if revumenib proves to be largely effective against these cancers, it may not last long for some. In a second study looking at the study data, also published this week in Nature, the researchers found that some patients’ cancers adapted to the treatment and developed resistance to it.
These findings are both good and bad news, say the authors. It is well, because it shows that these cancers really do depend on menin to drive their growth and likewise that drugs that can interfere with these cancers’ use of the protein can castrate them. At the same time, it probably also means that other strategies will be needed to delay or prevent resistance to these drugs. That could include treating people at an earlier stage of cancer or combining revumenib with other types of drugs that attack these cancers from a different angle.
An expansion of the study is already underway and full Phase I/II results are expected later this year. If things continue to go well, the company has said it may begin filing for FDA approval as early as late 2023. If approved, revumenib would be the first menin inhibitor to treat cancer.