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A new drug is showing promise in treating two types of leukemia. Abdullah Durmaz/Getty Images
  • Researchers report that the experimental drug revumenib showed promise in an early clinical trial in treating two types of leukemia.
  • They said 18 people with acute myeloid leukemia or acute lymphocytic leukemia achieved complete remission using the drug.
  • The clinical trials for revumenib are continuing.

Leukemia can be a difficult cancer to treat.

Among the most challenging types of leukemia are acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL).

People with these types of leukemia often endure a variety of treatments but continue to struggle to find one that gives them durable remissions.

However, a new drug that is still in clinical trials is showing promise in providing complete and durable remissions in both adults and children.

The results of a phase 1 trial of 60 people with aggressive leukemia whose published in the journal in Nature showed that more than half of those enrolled responded to the new drug, which is called revumenib.

In all, 18 people achieved complete or near-complete remission.

A targeted therapy that comes in pill form, revumenib works by inhibiting the activity of a protein called menin, which scientists say plays an important role in certain forms of leukemia.

These cancers carry a gene mutation or a rearrangement of another gene. The abnormalities that follow are commonly found in both pediatric and adult cases of these leukemias.

The drug breaks up that interaction.

Trials for the revumenib are continuing at the University of Texas MD Anderson Cancer Center, the City of Hope in California, and other leading cancer centers, the study authors noted.

Dr. James Mangan is a hematologist at the University of California San Diego.

He specializes in treating people with blood cancers, including acute lymphocytic leukemia, acute myelogenous leukemia, chronic lymphocytic leukemia, and chronic myeloid leukemia.

Mangan, who is not involved in the clinical trials, said the drug revumenib has great promise.

“Revumenib is a very exciting new drug. It’s a breakthrough medicine because it actually is a targeted therapy with broad downstream effects. And it is effective in large populations of people with AML. We have never been able to target this before,” Mangan told Healthline.

Michael Metzger is the chief executive officer of Syndax Pharmaceuticals, the company that is developing revumenib.

He told Healthline that the company is focused on this pivotal trial with the hope that it will result in a submission to the Food and Drug Administration (FDA) and approval of revumenib in 2024.

“We want to get this drug to the most people we can. And we want to get it to more babies and children who suffer from these types of leukemias and have few or no other options,” Metzger said.

“Pediatrics are the key to getting this drug to more people and to getting it FDA approved. That is our focus,” he added.

Menin is primarily a nuclear protein, but lesser amounts are also detectable in the cytoplasm and even the cell membrane.

Menin acts as a tumor suppressor, which means that it keeps cells from growing and dividing too quickly or in an uncontrolled way.

Although the exact functions of menin are unclear, it is likely involved in several important cell functions.

Dr. Gwen Nichols, the chief medical officer of the Leukemia & Lymphoma Society, told Healthline that menin inhibitors have shown benefits in treating both adults and children with these subtypes of acute leukemia.

These are super-interesting agents. We are looking at several companies for two of our clinical trials: the Beat AML trial as well as for the PedAL pediatric trial,” she said.

Revumenib was granted Orphan Drug Designation by the FDA and the European Commission for the treatment of people with AML.

It also received Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation.

Though nearly all of the participants in the latest trial experienced adverse 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 undergone several previous treatments, this is a very encouraging result,” said Dr. Scott Armstrong, a study author and president of the Dana-Farber Cancer Institute and Boston Children’s Cancer and Blood Disorders Center, in a press statement.

The finding provides “formal proof in patients that menin itself is a valid target for therapy in both genetic subtypes of AML,” Armstrong said.