- Posted June 5, 2026
Two-Pill Combo Offers Hospital-Free Leukemia Treatment Option For Older Adults With AML
An already available two-pill combo treats leukemia just as well as IV drugs, which should make cancer treatment easier on older adults, a new clinical trial has concluded.
The combo – decitabine-cedazuridine and venetoclax – showed strong response rates and survival outcomes among older adults with acute myeloid leukemia (AML), researchers reported June 3 in The New England Journal of Medicine.
The U.S. Food and Drug Administration (FDA) approved this drug combo in May for the treatment of AML in adults 75 and older or patients unable to withstand traditional chemotherapy, researchers noted.
Decitabine-cedazuridine (Inqovi) has been available in the United States since 2020, when the FDA approved it for treatment of another group of blood cancers called myelodysplastic syndrome.
“Having received approval, we anticipate that this oral AML regimen will become the standard of care for patients who are older or more frail,” said lead researcher Dr. Gail Roboz, director of the Clinical and Translational Leukemia Program at Weill Cornell Medicine in New York City.
Because patients only have to take pills, they won’t have to make repeated trips to a hospital or clinic for IV treatment, researchers said.
“We hope these results point to a future for AML patients where the treatment journey is less disruptive and less burdensome without sacrificing outcomes,” Roboz said in a news release.
AML is an aggressive blood cancer that is especially difficult to treat in older adults, researchers said in background notes.
Current standard treatment combines the pill venetoclax with a class of drugs known as hypomethylating agents, researchers said. Venetoclax inhibits a protein that leukemia cells overproduce to avoid dying off, while hypomethylating agents help slow leukemia progression by promoting healthy cell growth and survival.
However, hypomethylating agents must be administered in monthly cycles, requiring patients to spend five to seven days each month receiving IV drugs in a medical setting, researchers said.
Decitabine is one of those hypomethylating agents, and pharmacologists recently created a pill version by combining it with another drug called cedazuridine. The added drug keeps decitabine from being broken down when ingested.
To test whether this pill version of decitabine works when combined with venetoclax, researchers recruited 189 newly diagnosed AML patients being treated in the United States, Canada and Spain. Here's what they found:
All patients regularly took venetoclax, combined with five days of the decitabine-cedazuridine pill at the start of each treatment cycle.
Nearly half of patients (47%) had a complete response to the drug, with all signs of cancer disappearing.
About 63% experienced either a complete response or a complete response in which their healthy blood cell counts had not returned to normal.
Median overall survival reached nearly 16 months, comparable to existing IV therapies. (Median means half survived longer, half for a shorter time.)
Side effects were about the same as with IV therapies, most commonly a severe decline in healthy red and white blood cells.
Researchers recommend carefully monitoring leukemia cells until they’ve reached a certain threshold and then strategically pausing venetoclax, to allow the body to replenish normal blood cells.
“The goal of the all-oral therapy is to keep people out of the hospital, especially once they have achieved remission,” Roboz said. “Patients are thrilled not to have to deal with monthly chemotherapy injections or infusions.”
This combo isn’t a cure, researchers said. Patients must continue treatment to remain in remission.
“AML patients taking ongoing cycles of treatment require close monitoring but can still have an excellent quality of life,” Roboz said.
Researchers are now exploring what they call “triplet therapies,” in which targeted drugs will be added to the two-pill combo.
“The goal is to get away from treatment cycles that go on indefinitely,” she said. “We want to drive the leukemic cells to such low levels that patients can discontinue therapy and be cured.”
The study was funded by Taiho Oncology, the maker of Inqovi.
More information
The American Cancer Society has more on acute myeloid leukemia.
SOURCE: Weill Cornell Medicine, news release, June 3, 2026
