Cancer drug pushes mutant blood stem cells to become short-lived immune cells

The biologic drug interferon-alpha can benefit patients with blood cancers called myeloproliferative neoplasms by forcing mutant blood stem cells to become shorter-lived white blood cells, according to a study by Weill Cornell Medicine investigators. Because the broad activity of interferon-alpha can induce significant side effects, developing more focused strategies based on these mechanistic findings could meet an important need in cancer therapy.

Myeloproliferative neoplasms arise when DNA mutations in blood stem cells lead to the excess production of specific types of blood cells, such as megakaryocytes, which make platelets. Interferon-alpha often helps patients by reducing these imbalances and depleting the pool of mutant blood cells.

In the study, published in Nature Genetics, the investigators used advanced single-cell profiling tools to discover how interferon-alpha exerts these effects.

“These findings provide strategies for new ways to manage these and potentially other blood cancers,” said study senior author Dr. Anna Nam, an assistant professor of pathology and laboratory medicine and a member of the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine and a pathologist at NewYork-Presbyterian/Weill Cornell Medical Center.

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