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New drug may be very effective in treating AML

Image credit – Healthy Children (American Academy of Pediatrics)

CANCER DIGEST – March 18, 2023 – An early trial of a new immunotherapy drug showed that 53 percent of patients with acute myeloid leukemia (AML) responded to the drug, and 30 percent had a complete response to the drug, meaning all signs of cancer disappeared. However, there was a catch, after the second cycle of treatment, some patients showed signs of developing resistance to the drug.

Thee research teamed led by Dr. Scott Armstrong of the Dana Farber Cancer Institute, said the trial offered good news and bad news for AML patients.

"For patients with acute leukemia who have undergone several previous treatments, this is a very encouraging result," Armstrong said in a press release. "However, after the second cycle of treatment, some patients did develop resistance to revumenib."

AML is a cancer of blood and the bone marrow. Acute means the cancer tends to get worse quickly. The two subtypes of AML in this study accounts for about 40 percent of AML in children and adults.

The AUGMENT-101 trial is a phase 2 clinical trial designed to define the safety and effectiveness of the drug revumenib, an immunotherapy drug the specifically targets menin a protein involved in the process of switching gene activity on and off to regulate cell growth. When menin is mutated it fails to regulate that process and a variety of cancers of the hormonal system as well as AML can result.

In a follow-up study, the researchers found that the patients who began to develop resistance to revumenib had cancer cells that mutated to create a deformed version of menin that doesn’t bind with revumenib, blunting its effectiveness. That finding serves to confirm that menin is an effective target for therapy in AML. The two studies were published in the March 15, 2023 journal Nature.

For now, patients that don’t develop resistance, revumenib offers an additional therapy that could prolong survival. For patients that develop resistance to the drug, the researchers are already looking for ways to combine revumenib with other therapies to overcome the resistance.

The AUGMENT-101 trial is still enrolling patients. If you are interested in learning more about participating, ask your doctor about enrolling in AUGMENT-101. Physicians can contact the trial sponsor directly at

Sources: Dana Farber Cancer Institute press release


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