Leukemia Patient's Legacy Helps Drive Innovative Cancer Clinical Trial

RYE BROOK, N.Y., March 21, 2018 /PRNewswire-USNewswire/ -- Turning sorrow into action, The Harry T. Mangurian Jr. Foundation, whose namesake died from acute myeloid leukemia (AML), continues to fuel The Leukemia & Lymphoma Society's (LLS) ambitious attack on this deadly blood cancer.  Specifically, with the Foundation's support, LLS is leading a paradigm shift to treating cancers with a precision medicine approach – the right drug for the right patient at the right time – through its Beat AML® Master Clinical Trial.

The Beat AML Master Clinical Trial, launched in 2016, is a major collaboration bringing together multiple pharmaceutical companies, top cancer centers and scientists, the U.S. Food and Drug Administration, and a leading-edge genomics company, to bring novel, targeted therapies to patients faster. The trial is proceeding rapidly, with more than 200 patients enrolled in 10 study arms at eight prestigious cancer centers, including Memorial Sloan Kettering Cancer Center, The Ohio State University and OHSU Knight Cancer Institute.  More than 20,000 Americans are diagnosed with AML each year, and 10,000 AML patients die each year. There have been few advances in treatment for this disease in more than 40 years.

The Foundation, which previously donated $9 million to support LLS's Beat AML initiatives, has now committed an additional $10 million to help LLS push its pursuit of AML cures further, and help expand the trial by adding more study arms.

"This significant gift from The Harry T. Mangurian Jr. Foundation has the power to transform and will accelerate LLS's efforts to advance the science and treatment for patients with this deadly cancer, by revolutionizing how AML is studied and treated," said Louis J. DeGennaro, Ph.D., LLS president and CEO. "The generosity of the Mangurian Foundation, and their long-term commitment to our quest to cure AML cannot be overstated.  Not only is it a testament to their confidence in us to make a real impact on patients, but it demonstrates the value of  'investing' with LLS as our efforts are paying off for patients today. We need this kind of support now, more than ever, as sources of funds for innovative medical research are harder to come by."

Meeting a Critical Need

It is the fate of patients like Harry T. Mangurian Jr., a philanthropist who operated a race horse breeding farm in Florida and once owned the Boston Celtics, but lost his battle to AML in 2008, that inspires LLS to go on the offensive against this aggressive blood cancer.  Mangurian's experience is similar to many others diagnosed with this disease; lack of progress and options for treatment, particularly for older patients who cannot tolerate the standard chemotherapy regimen. Overall prognosis remains poor, with a five-year survival rate below 20 percent for patients older than age 60.

"We recognize that LLS is uniquely positioned among cancer organizations to actually make a difference in AML, and we are hopeful that our support of this wonderful organization will help speed new treatments and cures for the thousands of patients battling this horrible disease. In honor of our founder, Harry T. Mangurian, Jr., we must find cures," said its spokesperson and president Stephen G. Mehallis. 

A More Efficient Model

DeGennaro points out that the collaborative Beat AML Master Clinical Trial will stand as a model for how cancer clinical trials can be conducted in the future.

"By testing multiple drugs simultaneously, requiring fewer patients for each study arm, a quick timeline for accruing patients and testing the therapies, and a nimble protocol that allows new drugs to be added or removed quickly if they are not working, the trial is designed not only to bring new therapies to patients faster, but to operate in an more cost-efficient manner," DeGennaro said.

The Power of Collaboration

Recognizing the challenge this disease presents for patients, LLS is taking a comprehensive approach to tackling AML.   Along with the trial, LLS invests approximately one quarter of its research budget in AML science, and has extensive patient support and education programs.

The Beat AML Master Clinical Trial continues to grow, as another major cancer center, Huntsman Cancer Institute at University of Utah joined this month. They join Memorial Sloan Kettering Cancer Center, The Ohio State University Comprehensive Cancer Center, Knight Cancer Institute at Oregon Health and Science University, University of Texas Southwestern Medical Center, The University of Chicago Medicine Comprehensive Cancer Center, University of Colorado Anschutz Medical Center, and University of Maryland Marlene and Steward Greenbaum Comprehensive Cancer Center, bringing the total to eight centers. Four additional centers are poised to join in the coming months. 

Further, Agios Pharmaceuticals is the latest pharmaceutical company to provide its investigational drug to the study arms, bringing the total number of companies to seven; there are 10 active treatment arms, as one arm is for patients who don't have one of the identified genetic markers in the trial, and several of the companies' drugs are in more than one arm. The other companies in the collaboration are Alexion, Boehringer Ingelheim, Celgene, Gilead, Astellas and Takeda. 

The companies are providing the following investigational drugs: Agios: ivosidenib (IDH1 inhibitor); 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: pevonedistat (NAE inhibitor).

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 genetic analysis is being completed in seven days, a timeframe that is unprecedented in genomic technology.

"To date, this trial has exceeded our expectations in terms of collaborators and patients participating, a testament to the critical need for new approaches to treating this formidable foe," DeGennaro said.  "With help from the Mangurian Foundation and other supporters, we will continue to drive the development of precision medicine forward."

To learn more about the Beat AML Master Clinical Trial visit www.lls.org/beataml.

Media Contact: Andrea Greif ([email protected], 914-821-8958, 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 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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SOURCE The Leukemia & Lymphoma Society

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