The National Institutes of Health (NIH) has launched myeloMATCH, a precision medicine clinical trial designed to test new treatment combinations targeting specific genetic changes in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Funded by the NIH's National Cancer Institute (NCI), the trial aims to accelerate the discovery of tailored treatments for these aggressive blood and bone marrow cancers.
Precision Medicine Approach
myeloMATCH employs a unique approach by enrolling patients from initial diagnosis through multiple stages of their treatment. Newly diagnosed participants undergo rapid genetic testing of their tumor samples. Results are obtained within 72 hours, which allows researchers to match patients to sub-studies testing treatments appropriate for the specific genetic changes and characteristics associated with their disease. If a suitable sub-study is unavailable, patients receive standard-of-care treatment and are re-evaluated later for potential trial eligibility.
Richard F. Little, M.D., NCI coordinator for myeloMATCH, emphasized the importance of this approach: "AML and MDS are a heterogeneous group of cancers that can progress very quickly. Treatment advances depend in part on the ability to rapidly identify which subtype of cancer each patient has so that treatments can be tested for their specific cancer. The goal of myeloMATCH is to test combinations of drugs to treat the disease in a highly targeted way and to be able to start treatment quickly after diagnosis."
Trial Design and Objectives
The myeloMATCH trial is structured around randomized phase 2 trials, with each arm generally including 25 to 60 participants. Researchers expect results within 1 to 3 years, using early endpoints to identify the relative effect of treatments among the arms. This informs the most promising approaches for more definitive development.
The trial aims to enroll several thousand people within the first few years, with new sub-studies rolled out over time. Blood and bone marrow samples collected from participants will be used to develop and refine assays, as well as understand what genetic changes might be associated with treatment resistance.
Collaborative Network and Infrastructure
myeloMATCH is being conducted by NCI's National Clinical Trials Network, with participation from the NCI Community Oncology Research Program (NCORP). Initial sub-studies are led by research groups including the SWOG Cancer Research Network, the Alliance for Clinical Trials in Oncology, the ECOG-ACRIN Cancer Research Group, and the Canadian Cancer Trials Group. Clinical laboratory support is provided by the Frederick National Laboratory for Cancer Research, Fred Hutch Cancer Center, and Children’s Hospital Los Angeles.
Olatoyosi Odenike, MD, chair of the myelodysplastic clinical group for myeloMATCH and director of the leukemia program at University of Chicago, highlighted the accessibility of the trial: "The NCI National Clinical Trials Network is a huge network, which also includes community sites. It’s amazing to be able to actually bring this kind of 21st century precision medicine effort very close to where patients may live, and thereby really provide access to many who might not otherwise been able to access those kinds of efforts."
Building on Previous Success
myeloMATCH is one of three next-generation precision medicine trials that NCI has underway, following NCI-MATCH, which demonstrated the benefit of genomic sequencing in planning cancer treatment. ComboMATCH is testing new drug combinations for relapsed solid tumors, while ImmunoMATCH is assessing if immune status characterization can improve response to targeted immunotherapy treatments.
Danielle Carnival, deputy assistant to the President for the Cancer Moonshot, stated, "To achieve that goal, the U.S. government is working to expand access to innovative trials like this one, bring targeted new treatments to patients, and, ultimately, save lives."