The Leukemia & Lymphoma Society Achieves New Milestones in Offensive Against Acute Myeloid Leukemia, Deadly Blood Cancer

Health, Medicine

LLS Announces Progress in Groundbreaking Precision Medicine Clinical Trial and Significant Investment in New AML Research

The Leukemia & Lymphoma Society®(LLS) continues its assault against acute myeloid leukemia (AML), a deadly blood cancer that kills more than 10,000 Americans a year. LLS today announced its groundbreaking precision medicine Beat AML® Master Clinical Trial is seeing a rapid increase in patients enrolled as more leading cancer centers and biopharmaceutical companies join the collaboration. More than 150 patients are now enrolled in this innovative trial which aims to find new treatments for a blood cancer that has not seen significant treatment improvements in almost 40 years. Seven cancer centers, including Memorial Sloan Kettering Cancer Center (MSK) and the Knight Cancer Institute at Oregon Health and Science University (OHSU), are now enrolling patients.

The Need is Urgent

Each year, more than 21,000 Americans are diagnosed with AML, a rapidly progressing disease that remains one of the most lethal blood cancers. In fact, blood cancers are the third leading cause of cancer deaths in the United States. Despite advances in treating other blood cancers, the standard of treatment for AML – a combination of toxic chemotherapies – has changed very little over the past four decades. Overall prognosis remains poor, with a five-year survival rate of approximately 26 percent for patients, which decreases with age.

As the world’s largest nonprofit health organization dedicated to blood cancers, LLS is leading the charge against AML to help deliver new treatments that will address this urgent, unmet medical need. The Beat AML Master Clinical Trial is giving patients therapies based on their genetic markers – which is a hallmark of this promising precision medicine approach.

“For too long we’ve treated AML as a one-size-fits-all disease when it is really a much more complex grouping of multiple subtypes of blood cancer, and this trial sets out to change that paradigm,” said Louis J. DeGennaro, Ph.D, LLS’s president and CEO. “Our goal with this innovative trial is to deliver the right drug to the right patient at the right time, and we’re encouraged by the progress we’ve seen in the past year.”

In addition to the Beat AML Master Clinical Trial, as part of its comprehensive attack on AML, LLS has also announced a commitment of more than $8 million in new scientific research to discover novel approaches to treating the disease. Among the new projects is a major $5 million, five-year multidisciplinary effort led by Steven Nimer, M.D., at Sylvester Comprehensive Cancer Center, University of Miami working with other experts at Johns Hopkins University and Memorial Sloan Kettering Cancer Institute to develop therapies targeting the mechanisms that switch genes on and off, and which frequently become abnormal in myelodysplastic syndromes (MDS) and AML.

Another important new project is led by Shannon Oda, Ph.D., working in the laboratory of Philip Greenberg, M.D., at Fred Hutchinson Cancer Research Center in Seattle, who is examining new ways to enhance cutting-edge T-cell immunotherapy for AML.

What’s new in LLS’s efforts against AML

More Centers and Pharmaceutical Companies Participating

The most recent cancer center to join the Beat AML Master Clinical Trial is University of MarylandMarlene and Stewart Greenebaum Comprehensive Cancer Center. Maryland joins some of the most prestigious medical centers across the country including MSK, Ohio State University Comprehensive Cancer Center (OSUCCC), OHSU Knight Cancer Institute, Harold C. Simmons Comprehensive Cancer Center at University of Texas Southwestern; University of Chicago Comprehensive Cancer Center; and University of Colorado Cancer Center. Several more cancer centers are expected to join in early 2018.

Two more pharmaceutical companies, Astellas and Takeda, recently joined Alexion, Boehringer Ingelheim, Celgene, and Gilead Sciences, each investigating targeted therapies to treat AML patients based on their subtype. There are now nine active treatment arms, each focused on a specific subtype of AML. The companies are providing the following investigational drugs:  Alexion: samalizumab/ALXN6000 (anti-CD200); Boehringer Ingelheim: BI 836858 (anti-CD33); Celgene: enasidenib/AG-221/CC-90007 (IDH2 inhibitor); Gilead: entospletinib (SYK inhibitor), Astellas: gilteritinib (FLT3/AXL tyrosine kinase inhibitor); Takeda: evonedistat (NAE inhibitor). Only enasidenib has been FDA approved, but as a treatment for relapsed/refractory patients, while it is being tested in a first-line setting in the Beat AML Master Clinical Trial.  None of the others are approved yet for any indications.

Leading Unprecedented Collaboration

One of the unique aspects of the Beat AML Master Clinical Trial is that it is led by a nonprofit health organization.

As a “patients first” organization, LLS is well-positioned to convene this vast collaboration of stakeholders within the cancer ecosystem, including major cancer centers, pharmaceutical companies and several technology companies helping to manage the trial. The trial protocol employs advanced genomic technology performed by Foundation Medicine to identify the cancer-driving genetic mutations in newly diagnosed patients age 60 and older, in order to match them with an investigational drug best suited to their subtype of AML. For this trial, the process is being completed within seven days, a timeframe that is unprecedented in genomic technology.

The Beat AML Master Clinical Trial is expected to eventually include 500 patients and will continue for at least two years at between 15 and 20 clinical sites.

Finally Seeing Progress

Despite progress in treating other forms of blood cancer, AML had suffered through a 40-year drought in new treatments until earlier this year, when four therapies finally broke through to achieve FDA approval. LLS’s investment assisted each of these somewhere along their development pathway. These include:

  • gemtuzumab ozogamicin (Mylotarg) for treatment of adults with newly diagnosed CD33-positive relapsed/refractory AML
  • midostaurin (Rydapt®) in combination with chemotherapy for a subset of patients with a mutation called FLT3
  • enasidenib (IDHIFA®) for AML patients with the IDH2 mutation
  • CPX-351 (Vyxeos), an innovative reformulation of two standard chemotherapies for patients with secondary AML, a high-risk subtype

“Since our founding almost 70 years ago, we have recognized the urgency of finding new and better treatments for patients diagnosed with AML,” DeGennaro said. “After four decades and millions of dollars invested, we are finally seeing results for AML patients that give us real reason for hope.”

The American Society of Hematology (ASH), the premiere medical society dedicated to hematologic malignancies and other blood disorders, has partnered with LLS to raise awareness about LLS’s Beat AML initiative to its more than 16,000 members.

To learn more about the Beat AML Master Clinical Trial visit

Media Contact: Andrea Greif (andrea.greif@lls.org914-821-8958, mobile: 914-772-3027)

About The Leukemia & Lymphoma Society

The Leukemia & Lymphoma Society® (LLS) is the world’s largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin’s disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.

Founded in 1949 and headquartered in Rye Brook, NY, LLS has chapters throughout the United Statesand Canada. To learn more, visit Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.