National Collaborative Precision Medicine Trial to Treat Acute Myeloid Leukemia Launched

October 19, 2016

COLUMBUS, Ohio -- The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) will play a key leadership role in a groundbreaking, collaborative clinical trial for acute myeloid leukemia (AML) spearheaded by the Leukemia & Lymphoma Society.

Known as “Beat AML,” this unprecedented clinical trial is collaboration between top leukemia researchers, biopharmaceutical companies and a leading genomics information company aimed at advancing treatment for AML, a deadly disease that affects 20,000 Americans annually but that has seen few treatment improvements in the past 40 years.

John C. Byrd, MD, D. Warren Brown Chair of Leukemia Research and hematology division director at the OSUCCC – James serves as co-principal investigator for the Beat AML trial. The local arm of the trial will be led by Alice Mims, MD, and William Blum, MD, of The OSUCCC – James.

Beat AML was announced on Monday, Oct. 17, 2016, by Vice President Biden, along with many new, Moonshot inspired initiatives across industry, non-profit and government.

AML is the most lethal of the blood cancers, which together are the third leading cause of cancer deaths in the United States. AML is responsible for more than 10,000 deaths each year. Despite advances in treating other blood cancers, the standard of treatment for AML – a combination of toxic chemotherapies – has remained the same for more than 40 years. Overall prognosis remains poor, with a five-year survival rate below 20 percent for patients over age 60.

Byrd says the Beat AML master trial is an incredibly exciting endeavor focused on changing how we approach treatment for AML patients.

“Currently, patients are often diagnosed with one type of leukemia – AML – and then quickly directed to a line of treatment without the full benefit of knowledge about the underlying genomic complexities of their disease. Patients are often in the hospital for weeks, very sick  – and at the end they are given the choice of stem cell transplant or to continue aggressive chemotherapy with the expectation that their disease cannot be cured,” explains Byrd.

“This trial represents a shift away from that paradigm. What we know now it that AML is not one disease but possibly a dozen subsets of the disease and genomic knowledge of that person's specific cancer is critical knowledge for directing treatment. Within seven days, patients on the Beat AML trial receive information about the type of AML they have and they are then assigned to a therapy that fits their type of leukemia.”

The Beat AML protocol will employ comprehensive genomic profiling (CGP) to find and match specific AML genetic mutations in newly diagnosed patients over age 60, with an investigational drug or drug combination potentially best suited to attack the specific molecular mutations causing the cancer.

According to Louis J. DeGennaro, PhD, LLS President and CEO: “The Leukemia & Lymphoma Society is uniquely qualified to lead this unprecedented clinical trial collaboration, rare for a non-profit and a first for LLS.  Beat AML, as we have named this Master Trial, showcases LLS’s stature in the cancer ecosystem,” he explained. “It demonstrates our ability to convene the medical and research communities to think and act boldly in the quest for new and better treatments for blood cancer patients, and our aim to accelerate the rate at which precisely targeted breakthrough therapies reach the patients who urgently need them.”

Beat AML is a Unique Approach

The protocol for the Master Trial is unique in these key ways:

  • The Beat AML Master Trial is among the first cancer clinical trials to be led by a nonprofit health organization. As a neutral party, LLS eases the way for multiple pharmaceutical companies to bring their drugs into the collaboration, allowing for different subtypes of AML to be treated simultaneously. Having a single master protocol will allow for a more efficient, nimble process and allows the protocol to adapt rapidly as new drugs enter and others leave the trial.
  • Patients will be newly diagnosed, in contrast to most AML trials designed for relapsed/refractory patients. AML patients who relapse from their treatment tend to have far more genetic mutations, so identifying the genetic mutations early may offer a better chance for successful treatment.
  • Trial design ensures both speed and sophistication between diagnosis and treatment. Typically, most AML patients are rushed into standard treatment immediately upon diagnosis.  In the Beat AML Master Trial, newly diagnosed patients will have their genomic data analyzed within seven days so they can be entered into the appropriate study arm. LLS and the American Society of Hematology are working closely to educate physicians about the safety and importance of this innovative Master Trial.
  • To ensure the patient’s voice is heard, the trial protocol also will include patient-reported outcomes as a measure of success, which is a recommendation of the White House Cancer Moonshot Blue Ribbon Panel Report.

A Collaborative Approach to Precision Medicine

In conjunction with the LLS research team led by Amy Burd, Ph.D., Beat AML showcases the strong commitment to collaboration of leading AML experts, eager to put patients first. Lead investigators include John Byrd, MD, The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute; Brian Druker, M.D., Oregon Health and Science University; and Ross Levine, M.D., Memorial Sloan Kettering Cancer Center, all of whom have worked closely with LLS to plan and design the master protocol for the trial.

The trial will launch initially at five leading cancer centers – The Ohio State University Comprehensive Cancer Center, Memorial Sloan Kettering Cancer Center, Oregon Health and Science University, Dana-Farber Cancer Institute and Massachusetts General Hospital Cancer Center and start with four treatment “arms.” The first patients are expected to be enrolled by December 2016. Six additional clinical sites are prepared to begin enrolling patients in April, and the trial will eventually expand to between 15 to 20 sites, and up to 10 different treatment arms. LLS anticipates that 500 patients will be treated in the Master Trial. Once enrolled, trial duration for patients will range from one to three years.

The following four biopharmaceutical companies – Alexion, Boehringer Ingelheim, Celgene, and Gilead Sciences - are participating in the Beat AML Master Trial, offering the following investigational drugs, respectively: samalizumab (ALXN600), BI 836858, enasidenib, entospletinib. Many other pharmaceutical companies have expressed interest in joining the study, and additional treatment arms may be added over time.

Other Beat AML Master Trial collaborators include: Foundation Medicine, a leading genomics information company, which will utilize its proprietary comprehensive genomic profiling assay  for all of the patients; INC Research, a clinical research organization, which is helping LLS manage the logistics of the trial; Protocol First, providing a novel web-based digital application to help guide the clinicians; and MyClin, providing a solution to facilitate communication between the clinical trial sites.

LLS: A History of Leading the Fight Against AML

As the world’s largest voluntary health agency dedicated to fighting blood cancer, LLS has been funding AML research since our inception in 1949. Approximately 26 percent of LLS’s current research budget is invested in AML research. Beyond the Beat AML Master Trial, LLS has invested nearly $100 million over the past five years alone to better understand and treat this disease. For example, LLS had the vision to make a “down payment” for patients with AML in 2009 by partnering with Celator Pharmaceuticals to advance its drug CPX-351, an innovative formulation of two existing therapies, which recently outperformed standard therapy in a Phase 3 trial of patients with secondary AML, a high-risk subset of the disease. This investment has the potential to become the first new treatment approved in the US for AML patients in 40 years.

“Throughout our 67-year history, LLS has time and again taken a leadership role when it has recognized a challenge for patients. We’re doing it again with the Beat AML initiative, working together to advance new therapies and improve outcomes for these patients who desperately need new and better options,” said DeGennaro.


LLS Contact: Andrea Greif,, 914-821-8958

OSUCCC – James Contact: Amanda Harper,, 614-685-5420

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