Rye Brook, NY (July 19, 2017) – The Leukemia & Lymphoma Society (LLS) today announced the expansion of its groundbreaking precision medicine Beat AML® Master Trial, to test new, investigational treatments for acute myeloid leukemia (AML), the central initiative of LLS’s comprehensive assault on this deadly blood cancer affecting 20,000 Americans annually.
Launched in October 2016, the master trial has exceeded expectations with more than 70 patients enrolled to date. Six leading cancer centers across the country, including Memorial Sloan Kettering Cancer Center (MSK), Ohio State University Comprehensive Cancer Center (OSUCCC) and Oregon Health & Science University (OHSU) Knight Cancer Institute, are participating in the master trial; four more are expected to join by the end of the summer. Given the urgency to find new treatments for AML, four major biopharmaceutical companies are providing investigational targeted therapies, with three more expected to join soon. The therapies are administered to patients based on their genetic markers – a hallmark of this promising precision medicine approach.
Blood cancers are the third leading cause of cancer deaths in the United States, and AML alone is responsible for more than 10,000 deaths annually. 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 below 20 percent for patients older than age 60.
According to Dr. Louis J. DeGennaro, Ph.D., LLS president and chief executive officer, “As the world’s leading non-profit dedicated to fighting blood cancers, LLS is uniquely able to convene the key stakeholders in cancer research to make the Beat AML Master Trial possible. Working closely with the U.S. Food and Drug Administration (FDA), renowned cancer researchers, medical centers and the pharmaceutical industry, we are seeking to change the way cancer research is conducted.”
The Beat AML trial protocol employs advanced genomic technology performed by Foundation Medicine to identify the cancer-driving genomic mutations in newly diagnosed patients age 60 and older, in order to match them with an investigational drug suited to their subtype of AML. For this trial, the process is being completed in seven days, a timeframe that is unprecedented in genomic technology.
“LLS is a patients first organization, and cancer patients have waited too long for better treatments for AML,” said DeGennaro. “We are very grateful to those patients who have enrolled in the Beat AML Master Trial; the learnings and treatments resulting from this innovative trial will help tackle these diseases, by delivering the right drug to the right patient at the right time.”
LLS has been assisted in developing and managing the trial by three renowned physician scientists: Brian Druker, M.D., OHSU Knight Cancer Institute; Ross Levine, M.D., MSK; and John Byrd, M.D., OSUCCC. Dr. Byrd was recognized recently by LLS with the prestigious Return of the Child Award, honoring the major and lasting scientific contribution he has made to the better understanding, management and treatment of blood cancers.
Adding More Cancer Centers and Treatment Arms
Presently, six cancer centers are enrolling patients: OSUCCC, MSK, OHSU Knight Cancer Institute, University of Texas Southwestern, University of Colorado Cancer Center and University of Chicago Comprehensive Cancer Center. Four more are expected to join by the end of the summer.
The number of active treatment arms is now five, treating patients with drugs provided by Alexion, Boehringer Ingelheim, Celgene and Gilead Sciences. The companies are providing the following investigational drugs, respectively: samalizumab/ALXN6000 (anti-CD200); BI 836858 (anti-CD33); enasidenib/AG-221/CC-90007 (IDH2 inhibitor); entospletinib (SYK inhibitor). At least three more companies are expected to join soon. None of the therapies are approved yet for any indications.
The Beat AML Master Trial is expected to eventually include 500 patients and will continue for at least two years at between 15 and 20 clinical sites.
“We are very pleased with the patient enrollment to date and would like to offer our deepest thanks to our Beat AML Master Trial collaborators for their hard work and commitment to helping patients affected by this challenging disease,” said DeGennaro. “It takes a team to find cures and, together, we believe we can change the treatment paradigm for AML and bring new and better treatments to patients who urgently need them.”
Breaking New Ground
The Beat AML Master Trial is unique in that it is the first cancer clinical trial to be led by a non-profit organization. LLS was awarded the Investigational New Drug (IND) approval by the FDA and collaborates regularly with the regulatory body on the progress of the master trial.
The primary question the trial seeks to address is: Can a personalized therapy based upon genomics be assigned to an AML patient within a seven day period in a safe manner? Since its launch, the trial has already surpassed its first feasibility milestone of successfully screening more than 20 patients within seven days. In fact, that number has been far exceeded, with more than 70 patients treated to date.
A History of Leading the Fight Against AML
While the Beat AML Master Trial is the primary focus of LLS’s offensive against AML, the organization is leading the fight on many fronts. Approximately 26 percent of LLS’s current research budget is invested in AML research and LLS has committed resources to its patient services programs to help patients through education and support services. 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 (acquired by Jazz Pharmaceuticals in 2016) to advance its investigational drug CPX-351 (Vyxeos ®), an innovative formulation of two existing therapies. The drug outperformed standard therapy in a Phase 3 trial of patients with secondary AML, a high-risk subset of the disease and is currently under FDA review with the potential to become the second new treatment approved in the U.S. for AML patients in 40 years. An FDA decision on this therapy is expected by September.
The first new AML therapy approved in decades is midostaurin (Rydapt ®), FDA approved on April 28. This recent approval marked the first significant advance for AML patients in 40 years. Approved in combination with chemotherapy for a subset of patients with a mutation called FLT3, it is also among the first targeted therapies for AML. LLS provided substantial funding to several researchers involved in the preclinical and clinical work helping to identify midostaurin’s effectiveness in killing FLT3 AML cells. Another therapy for AML, gentuzumab ozogamycin (Mylotarg ®), received accelerated FDA approval in 2000 but was withdrawn from the market in 2010 because of its toxicity. On July 11, an FDA panel recommended bringing it back to market for newly diagnosed adult AML patients, in combination with standard chemotherapy. The FDA will decide by September.
“Throughout our 68-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.
To learn more about the Beat AML Master Trial visit www.lls.org/beataml or view our media toolkit.
Media Contact: Sandra Salviejo, (sandra.salviejo@lls.org, 516-659-7931)
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 States and Canada. To learn more, visit www.LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.
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