Rapid precision medicine is feasible in fight against a formidable leukemia

The Beat AML clinical trial has matched 250 study participants to targeted treatments for acute myeloid leukemia – with promising early results.

Regulators were wary of the complicated study proposed by the Leukemia & Lymphoma Society and cancer centers including the OHSU Knight Cancer Institute. The clinical trial design called for holding off treatment of newly diagnosed acute myeloid leukemia for up to a week to make time for gene sequencing to match participants, if possible, with drugs selected to target a vulnerability specific to their cancer.

“I have to say we had some concerns,” said Ann Farrell, M.D., director of the Food and Drug Administration’s Division of Hematology Products. “You’re going to wait seven days until you treat patients with AML? You know, we don’t want a high death rate from lack of therapy,” she said during a roundtable discussion at the American Society of Hematology annual meeting in December. “We scrutinized this trial and gave lots of comments… There was a lot of back and forth. And then it started up and running, and we held our breath.”

Now, two years after launching, the Beat AML clinical trial has screened more than 400 patients, assigned more than 250 to investigational treatments with promising early results, and firmly established the feasibility of a rapid precision medicine approach in patients with previously untreated AML.

“That’s real progress,” Knight Cancer Institute Director Brian Druker, M.D., told attendees of the roundtable discussion. “That’s helping patients in a way that we haven’t been able to before.”

In the future, the dynamically evolving trial may begin testing combinations of novel anti-cancer agents.


The Beat AML trial is enrolling newly diagnosed patients age 60 and older. Researchers use a bone marrow biopsy to obtain tumor cells for next-generation sequencing. Results are used to assign participants to one of the trial’s treatment arms. It opened in 2016 with three treatment arms but has expanded to 11 treatment arms testing seven novel anti-cancer agents. In the future, the dynamically evolving trial may begin testing combinations of novel anti-cancer agents.

“Going into this there was a fair bit of skepticism that you could enroll patients with an acute illness in a clinical trial and wait seven days before you started them on treatment, or even turn around the genomics in seven days,” Druker said. “We’ve done that in over 95 percent of the patients.”

Each year, more than 20,000 Americans are diagnosed with AML and 10,000 die from it. The five-year-survival rate for older adults is less than 20 percent, according to LLS. The standard treatment has been a drug combination established 40 years ago.

But since the start of 2017, the FDA has approved nine anti-cancer agents as new treatments for AML.

“We’re really starting to see that we can do a better job of matching patients to therapies, understanding who’s going to respond, who will become resistant and why they become resistant and actually modifying our therapies when people become resistant so we can actually see more durable responses,” Druker said.

A video recording of the complete roundtable is available to view online.

Further reading:

Initial Report of the Beat AML Umbrella Study for Previously Untreated AML: Evidence of Feasibility and Early Success in Molecularly Driven Phase 1 and 2 Studies, presentation by Amy Burd at the American Society of Hematology Annual Meeting (December 3, 2018)

Study of Biomarker-Based Treatment of Acute Myeloid Leukemia, ClinicalTrials.gov