October 19, 2018
Study involving hundreds of patient samples may reveal new treatment options for patients facing an aggressive form of leukemia
After more than five years and 672 patient samples, researchers have published the largest cancer dataset of its kind for a form of leukemia. The study, titled “Functional Genomic Landscape of Acute Myeloid Leukemia” and led by OHSU Knight Cancer Institute in Portland, was published in Nature. Representatives from 11 academic medical centers, including The University of Kansas Cancer Center, played key roles in collecting and analyzing the 672 samples of cancer cells from 562 patients. Pharmaceutical and biotechnology companies, 11 in total, also contributed to the effort, by supplying drugs for testing.
Acute myeloid leukemia, or AML, has a low survival rate: less than 25 percent of newly diagnosed patients survive beyond five years. Developing effective, targeted AML therapies has proved challenging because AML is not a single disease, but a disease of many types with gene mutations and chromosome abnormalities that vary patient to patient. Consequently, the standard of care has largely remained unchanged for the past 40 years.
The study is part of the Leukemia & Lymphoma Society’s Beat AML initiative, a large-scale collaboration launched in 2013 that brings together academic medical centers with large AML programs, patients and pharmaceutical companies to analyze the genomic causes of AML and identify new treatment options. The long-term goal of BEAT AML is to develop personalized treatments for each patient with AML based on their disease biology and specific mutation pattern.
“The goal of this first phase of the Beat AML initiative is to better understand the drivers for AML, which then help us figure out the best treatment for each individual patient. This program is a tremendous example of the innovation and commitment the Leukemia & Lymphoma Society provides to blood cancer patients,” said Scott Weir, PharmD, PhD, director of the University of Kansas Medical Center’s Institute for Advancing Medical Innovation.
“This publication represents the first step in demonstrating the capability of the Beat AML team to rapidly characterize leukemia gene mutations and drug sensitivity of patient samples collected in real-time,” said Tara Lin, MD, director of KU Cancer Center’s Acute Leukemia Program and medical director of the Clinical Trials Office. “This project was a remarkable undertaking and represents five years and the generosity of hundreds of AML patients who participated and contributed their samples to the effort.”
Armed with a comprehensive dataset, researchers can simultaneously test the response of a patient’s leukemia cells to different targeted drugs and combinations of drugs in hopes of determining which medication will be most effective in inhibiting mutations and genetic drivers of the disease. The result is individualized treatment for every AML patient.
The next phase of the project, called the Beat AML Master Trial, is a clinical trial for patients who have been recently diagnosed with AML. KU Cancer Center will be one of the sites treating patients. Patient diagnostic samples will be taken and profiled for chromosomal and genetic abnormalities, as well as drug sensitivities. Doctors receive the results in just seven days. Once the doctor and patient have the results of the predicted best treatment, they decide whether to pursue the new treatment on the BEAT AML trial or try a standard treatment option.
“This is an entirely new way of treating this disease. We are not treating a patient the same way we’ve done before, instead we are looking at each individual, and their specific disease characteristics, and tailoring a treatment exactly for them,” Dr. Lin said.