Beat AML Master Clinical Trial Showcases Paradigm Shift With Precision Medicine


Delaying treatment for up to 7 days so that genomic data can be utilized to inform a personalized treatment approach is safe, feasible, and can improve overall survival in patients with acute myeloid leukemia.

John C. Byrd, MD

John C. Byrd, MD

Delaying treatment for up to 7 days so that genomic data can be utilized to inform a personalized treatment approach is safe, feasible, and can improve overall survival (OS) in patients with acute myeloid leukemia (AML), according to results from the Beat AML Master clinical trial (NCT03013998) published in Nature Medicine.1

Results showed that the trial met its primary end point in that it demonstrated that a genomic analysis of leukemia cells can be performed within 1 week to detect disease subtypes; this provides patients, caregivers, and healthcare providers with sufficient time to reach an informed treatment decision without negatively impacting survival.2

In fact, the 30-day mortality rate was found to be less frequent, and OS was substantially prolonged in those who received investigational targeted therapies or combinations based on their testing results compared with those who received standard-of-care (SOC) treatment.

“The study shows that delaying treatment up to 7 days is feasible and safe, and that patients who opted for the precision medicine approach experienced a lower early death rate and superior OS compared with patients who opted for SOC,” John C. Byrd, MD, D. Warren Brown Chair of Leukemia Research of The Ohio State University, BEAT AML lead, and corresponding author of the study, stated in a press release.

“This patient-centric study shows that we can move away from chemotherapy treatment for patients who won’t respond or can’t withstand the harsh effects of the same chemotherapies we’ve been using for 40 years and match them with a treatment better suited for their individual case,” Byrd added.

In fall 2016, the Leukemia & Lymphoma Society (LLS) launched the Beat AML Master clinical trial in an effort to evaluate several targeted therapies in patients with newly diagnosed AML aged 60 years and older at a number of cancer centers throughout the United States.

A partnership established with Foundation Medicine, Inc allowed for the utilization of next-generation genomic sequencing to generate a rapid analysis of leukemia cells and determine disease subtypes. Based on the results from these tests, patients would then be assigned to a sub-study within the protocol to receive a targeted agent.3

To date, more than 1,000 patients have been screened in the trial. Results published in Nature Medicine focus on patients who were enrolled to the trial between November 17, 2016 and January 30, 2018. A total of 395 patients with suspected AML were determined to be eligible for inclusion during that timeframe. The median age of these patients was 72 years (range, 60 years–92 years) and 38% of patients were 75 years of age or older.

Of those patients, 374 (94.7%) successfully completed genetic and cytogenetic analysis within a 7-day timeframe. Ultimately, 224 of these patients, or 56.7%, opted to enroll on 1 of the 11 active study arms. The remainder of the patients (n = 171) either opted for standard of care (n = 103), palliative care (n = 40), or investigational therapy on an alternative clinical trial (n = 28). Nine patients died before they were assigned to a treatment.

Demographic, laboratory, and molecular characteristics were not found to substantially differ between participants enrolled to the sub-studies and those who opted to receive SOC treatment, which included induction with cytarabine and daunorubicin (7+3 or equivalent) or a hypomethylation agent.

The median OS in those who were enrolled to 1 of the targeted therapy study arms was 12.8 months versus just 3.9 months in patients who opted to receive a SOC treatment approach.

“The study is changing significantly the way we look at treating patients with AML, showing that precision medicine, giving the right treatment to the right patient at the right time, can improve short- and long-term outcomes for patients with this deadly blood cancer,” Louis J. DeGennaro, PhD, president and CEO of LLS, added in the release. “Further, Beat AML has proven to be a viable model for other cancer clinical trials to emulate.”


  1. Burd A, Levine RL, Byrd JC, et al. Precision medicine treatment in acute myeloid leukemia using prospective genomic profiling: feasibility and preliminary efficacy of the Beat AML Master Trial. Nat Med. Published online October 26, 2020. doi:10.1038/s41591-020-1089-8
  2. Study in Nature Medicine shows superior outcomes for patients in LLS’s paradigm-shifting Beat AML clinical trial. News release. Leukemia & Lymphoma Society. October 26, 2020. Accessed October 28, 2020.
  3. Study of biomarker-based treatment of acute myeloid leukemia. Updated September 18, 2020. Accessed October 28, 2020.
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