In a to study this week, scientists describe a new experimental pill that may be able to help patients with a difficult-to-treat form of leukemia. Almost half of the patients in the phase I trial responded to the treatment, called revumenib, while around a third experienced complete remission of their cancer. However, more research will be needed to confirm its effectiveness.
Revumenib is developed by the Syndax company. It is believed to work by inhibiting a protein called menin, which appears to play an important role in some forms of leukemia. These cancers are characterized by carrying a mutation of the NPM1 gene or a rearrangement of another gene, called KMT2A. These aberrations are commonly found in pediatric and adult cases of leukemia and are often very difficult to treat.. According research cited by scientists, for example, the five-year survival rate for KMT2A-related leukemia is less than 25%.
Thursday, these researchers published results of the first human trial of revumenib in the journal Nature. The trial recruited 68 patients with relapsed or refractory acute leukemia who had not responded 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 effectiveness, the team also looked at the results of people with NPM1/KMT2A-related leukemia (60 in total). Of these eligible patients, 53% showed some response to revumenib and approximately 30% (18 patients) showed complete remission. Although almost all patients experienced adverse effects, including potentially serious effects such as irregular heartbeat, none had to stop treatment as a result.
“For patients with acute leukemia who have had multiple prior treatments, this is a very encouraging result,” said study author Scott Armstrong, president of Dana-Farber and Boston Children’s Cancer and Blood. Disorders Center, in a statement 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 even if revumenib is largely effective against these cancers, it may not last long for some. In a second study Examining data from the trial, also published this week in Nature, the researchers found that some patients’ cancers were adapting to treatment and developing resistance to it.
These results are both good and bad news, according to the authors. It’s good, because it shows that these cancers really depend on menin to fuel their growth and also that drugs capable of disrupting the use of the protein by these cancers can neutralize them. At the same time, it also likely means that other strategies will be needed to delay or prevent resistance to these drugs. This could include treating people with early stage 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, with full Phase I/II results expected later this year. If things continue to go well, the company has said it could begin filing for FDA approval as early as late 2023. If approved, revumenib would be the first menin inhibitor to treat cancer.