The Leukemia & Lymphoma Society (LLS) and the Knight Cancer Institute at Oregon Health & Science University (OHSU) today announced a pioneering collaboration that brings together scientists from multiple disciplines to better understand a complex form of leukemia for which there are currently no broadly effective treatments.
The multi-institution Beat AML cancer research initiative – designed to leverage the expertise of technology, sequencing and pharmaceutical collaborators − takes a next-generation personalized medicine approach to vastly accelerate research findings and ultimately improve outcomes for patients with acute myeloid leukemia (AML). AML is a particularly devastating blood cancer with less than 25 percent of newly diagnosed patients surviving beyond five years. It causes more than 10,000 deaths a year in the U.S., and treatment options largely have not changed in the past 30 years.
LLS and the Knight Cancer Institute are forging Beat AML to change the paradigm of treatment for AML patients. The initiative acknowledges that AML is, in reality, a diverse collection of poorly understood rare diseases that share some common traits. Because of its complexity, improving prospects for AML patients requires a transformative approach that acknowledges the biological diversity across AML cases. The research process is designed to provide rapid analysis of the way in which genes malfunction in individual patients with the disease, how the disease progresses as well as how it responds to, or evades, treatment.
“This innovative collaboration – involving the world’s largest nongovernment funder of blood cancer research, a group of leading academic research institutions, two advanced technology companies and potentially multiple pharmaceutical and biotechnology companies – is among the first of its kind in the cancer space and unprecedented in terms of the range of expertise involved,” said Brian Druker, M.D., director of the Knight Cancer Institute at OHSU. “We are setting ambitious milestones with the goal of helping patients. The project also might help accelerate other cancer research by serving as a model for the types of collaboration that are possible to advance research.”
The project will be led by Druker and includes researchers at Stanford University, UT Southwestern Medical Center and Huntsman Cancer Institute at the University of Utah. Intel Corporation and Illumina are providing computational analysis and genetic sequencing expertise.
The three-year project also seeks to add more collaborators including pharmaceutical and biotech companies that will test a comprehensive offering of novel drugs to address the underlying molecular complexity of AML. As part of this effort, Array BioPharma will be the first biopharmaceutical company to evaluate its therapeutics with this project.
Beat AML will create a profile of the possible genetic drivers of AML by conducting a deep genomic sequencing analysis of participating AML patients’ samples. As information from the samples is analyzed by the Knight Cancer Institute’s bioinformatics team to determine potentially relevant mutations, researchers will simultaneously test the response of patients’ leukemia cells to different drugs and combinations of drugs. This dual process will better equip scientists to confirm that they have correctly identified a genetic driver of the disease. This type of approach not only speeds progress in understanding AML, but more efficiently determines ways to stop the disease and better block potential recurrence.
LLS has committed to investing more than $8.2 million in the initial three-year project which will analyze samples of cancerous cells from 900 patients with AML. The volume of samples analyzed and the level of detail collected will enable the Beat AML team to build an extensive biological map of the disease using their functional genomic approach. Researchers involved hope this enormous data set will lead to identification of potential new drug targets as well as novel combinations of drugs. The goal of the project is to move this information rapidly into the clinic by matching patients with treatments that target their leukemia more precisely.
Druker’s research, which has received significant support from LLS, has already revolutionized treatment of another form of leukemia, chronic myeloid leukemia (CML).
“Now we hope to do for patients with AML what has been achieved with CML: Take a blood cancer that was, with few exceptions, a death sentence, and enable patients not only to survive, but to enjoy a longer, richer quality of life,” LLS President and Chief Executive Officer John Walter said. “The Leukemia & Lymphoma Society is focused on finding cures and ensuring access to therapies for all blood cancer patients and our priority is to employ the best science to help us address critical unmet medical needs, which is why we are partnering with the OHSU Knight Cancer Institute, a leader in developing targeted cancer medicines, and Brian Druker’s team, whose track record of success is second to none.”
Druker helped prove it was possible to shut down the mutations driving cancer without harming healthy cells, helping to usher in the era of personalized cancer medicine with molecularly targeted therapeutic approaches. The drug Gleevec®, which was developed out of Druker’s early research, is now among one of dozens of similar drugs approved by the U.S. Food and Drug Administration (FDA).
Gleevec changed the average life expectancy for CML patients who previously could expect to live only about five years after diagnosis. Now they have a normal lifespan and quality of life.
Brian Druker, M.D., is director of the Oregon Health & Science University Knight Cancer Institute, associate dean for oncology in the OHSU School of Medicine, JELD-WEN Chair of Leukemia Research at OHSU, and a Howard Hughes Medical Institute investigator.