# Yale phase I-II trial of a two-pill regimen in 189 older AML patients: 47% complete response

- Published: 2026-11-12 06:04 UTC
- Section: Life & Health › Medicine
- Tags: acute myeloid leukemia, all-oral regimen, decitabine cedazuridine, venetoclax, ASCERTAIN-V, Yale Cancer Center, FDA approval, older patients, New England Journal of Medicine
- URL: https://realtech.media/news/rt-e9db9347f3acc0751355704e_yale_phase_i/

**In an indirect comparison, the two-pill regimen showed survival similar to injection-based treatment; the FDA has approved it for older patients with AML.**

![Yale phase I-II trial of a two-pill regimen in 189 older AML patients: 47% complete response](https://realtech.media/media/2026/11/4639350d1ee7469d.jpg)
*newswise.com*

Researchers at Yale School of Medicine and Yale Cancer Center have published in the New England Journal of Medicine a study of an all-oral regimen for older patients with newly diagnosed acute myeloid leukemia, according to medicine.yale.edu [1]. The phase I-II ASCERTAIN-V trial enrolled 189 patients who were 75 or older, or had health conditions that made intensive chemotherapy unfavorable [1]. Based on the results, the U.S. Food and Drug Administration (FDA) has approved the regimen for these patients [1].

## Replacing clinic injections with pills

Acute myeloid leukemia is an aggressive cancer of the blood and bone marrow. Until now, older patients have needed clinic injections five to seven days a month, plus a daily oral pill [1]. Amer Zeidan, professor of medicine at Yale School of Medicine and co-first author, said patients spend more than a quarter of their remaining life going to the clinic, and some choose to forgo treatment entirely [1].

The trial replaced the injection with two oral pills. One combines decitabine, a chemotherapy that slows cancer growth, with cedazuridine, an ingredient that lets decitabine work as a pill. The second is venetoclax, an inhibitor that blocks a protein cancer cells need to survive [1].

Once a patient's bone marrow was clear of cancer, the team shortened the dosing in later cycles. Venetoclax went from 28 days per cycle to 21, 14 or seven days, depending on how quickly the blood count recovered. Decitabine-cedazuridine went from five days per cycle to three to four days [1]. According to Zeidan, this allowed healthy blood counts to recover faster and reduced the risk of infections and bleeding [1].

## Trial results

After the all-oral regimen, 47% of patients reached a complete response, meaning no detectable signs of cancer remained. Another measure, a significant clinical response, was reached by 63% of patients [1]. Of those who had needed regular blood or platelet transfusions, 60% no longer needed them during treatment [1]. Median overall survival was 15.5 months, matching the traditional injection-based approach [1].

Zeidan noted that this is an indirect comparison, which found similar efficacy and safety to the standard regimen [1]. One patient, 83-year-old Pamela Young, has been in complete remission for almost three and a half years [1].

The team plans to use the two pills as a standard treatment and as a base for trials adding other targeted oral drugs [1]. Zeidan also leads a global study of 1,300 patients that combines another new therapy, ziftomenib, with standard therapies for acute myeloid leukemia [1].

## Sources

1. [medicine.yale.edu](https://medicine.yale.edu/breakthroughsreview/article/oral-therapy-allows-at-home-treatment-acute-myeloid-leukemia/)
2. [newswise.com](https://www.newswise.com/articles/oral-therapy-allows-at-home-treatment-for-acute-myeloid-leukemia)

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Source: RealTech, realtech.media — https://realtech.media/news/rt-e9db9347f3acc0751355704e_yale_phase_i/