NIH clinical trial will test precision medicine treatments for myeloid cancers

Oct. 29, 2024
Researchers will seek to accelerate the discovery of more tailored treatments for these aggressive cancers of the blood and bone marrow.

The National Institutes of Health (NIH) has launched a proof-of-concept precision medicine clinical trial to test new treatment combinations targeting specific genetic changes in the cancer cells of people with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

The trial, funded by NIH’s National Cancer Institute (NCI), aims to accelerate the discovery of more tailored treatments for these aggressive cancers of the blood and bone marrow.

Initially, people enrolled in the trial with newly diagnosed AML or MDS will undergo rapid genetic testing of their tumor samples. Based on the molecular characteristics of their tumors, they will be matched to a substudy testing a treatment appropriate for the specific genetic changes and characteristics associated with their disease, if one is available, or to standard treatment if an appropriate substudy is not available.

If the initial treatment works to reduce a patient’s disease, they will undergo further genetic testing to match them to a subsequent substudy testing a treatment that is appropriate for the specific genetic changes associated with their remaining disease. As the amount of a patient’s disease decreases, study investigators will use increasingly sensitive tools, such as biomarker assays, to identify appropriate treatments for any remaining cancer cells.

The myeloMATCH trial aims to enroll several thousand people within the first few years, with new substudies rolled out over time. Blood and bone marrow samples collected from participants during the course of the trial will be used to develop and refine the assays, as well as understand what genetic changes might be associated with the development of resistance to a treatment.

NIH release