Methotrexate (MTX), a medication used commonly in the treatment of arthritis, may be effective in patients with polycythemia vera (PV).

PV is a blood cancer characterized by bone marrow dysfunction that leads to an overproduction of red blood cells. Patients with PV may experience painful joints, itchiness, reddened face, weakness, and headaches. There are approximately 2 new cases of PV per 100,000 people annually

Researchers have discovered that the mechanism by which methotrexate works is by inhibiting the JAK/STAT signaling pathway, whose dysregulation is considered to be a key contributor to the development of several blood cancers collectively referred to as myeloproliferative neoplasms (MPNs); PV is one of the myeloproliferative neoplasms.

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For this study, researchers demonstrated that methotrexate acts as a potent inhibitor of the JAK/STAT pathway in human cells, and subsequent tests in mice models resulted in nearly identical results. The studies showed that low dose methotrexate normalized the increased blood counts and spleen size.

Dr Martin Zeidler, from the University of Sheffield’s Department of Biomedical Science, said, “Repurposing MTX has the potential to provide a new, molecularly targeted treatment for MPN patients within a budget accessible to health care systems throughout the world — a development that may ultimately provide substantial clinical and health economic benefits.”

A clinical trial assessing the efficacy of methotrexate in PV may be initiated next year.


1. Breakthrough by scientists finds arthritis drug could treat blood cancer patients [news release]. Sheffield, England: University of Sheffield; August 3, 2017. Accessed August 21, 2017. 

This article originally appeared on ONA