Scientists at the Wellcome Trust Sanger Institute in Cambridge, United Kingdom, and their international collaborators have shown that acute myeloid leukemia (AML) is not a single disorder, but at least 11 different diseases, and that genetic changes explain differences in survival among young AML patients. Published in the New England Journal of Medicine, the groundbreaking study on the genetics of AML could improve clinical trials and the way patients are diagnosed and treated in the future, according to the authors.
In the largest study of its kind, researchers studied 1,540 patients with AML who were enrolled in clinical trials. They analyzed more than 100 genes known to cause leukemia to identify common genetic themes behind the development of the disease.
The researchers found that the patients were divided into at least 11 major groups, each with different constellations of genetic changes and distinctive clinical features. Despite finding common themes, however, the study also showed that most patients had a unique combination of genetic changes driving their leukemia. This genetic complexity helps explain why AML shows such variability in survival rates among patients.
Full knowledge of the genetic make-up of a patient’s leukemia substantially improved the ability to predict whether that patient would be cured with current treatments. This information could be used to design new clinical trials to develop the best treatments for each AML subtype, with the ultimate aim of bringing more extensive genetic testing into routine clinical practice, according to the researchers.
AML is an aggressive blood cancer that affects people of all ages, often requiring months of intensive chemotherapy in a hospital. The disease develops in bone marrow cells.