Breakthrough by Sheffield scientists finds arthritis drug could treat blood cancer patients

Blood cancer sufferers could be treated with a simple arthritis drug, scientists at the University of Sheffield have discovered. bm

Every year 3,000 people in the UK are diagnosed with Polycythemia Vera (PV), a type of blood cancer which causes an overproduction of red blood cells. Patients suffer with itching, headaches, weight loss, fatigue and night sweats.

Current treatments do not slow the disease progression and provide little relief from symptoms.

Dr Martin Zeidler, from the Department of Biomedical Science in the Faculty of Science, working with colleagues from the Department of Haematology at the Royal Hallamshire Hospital, and funded by the Medical Research Council (MRC) have discovered that methotrexate (MTX) – a drug on the World Health Organisation list of essential medicines and commonly used to treat arthritis - works by directly inhibiting the molecular pathway responsible for causing disease.

Initial tests were carried out on fruit fly cells to screen for small molecules that modulate JAK/STAT signalling – a signalling pathway whose misregulation is central to the development in humans of Myeloproliferative neoplasms (MPNs), the collective term for progressive blood cancers like PV.

Further testing in human cells showed that methotrexate acts as a potent suppressor of JAK/STAT pathway activation – even in cells carrying the mutated gene responsible for MPNs in patients.

Dr Martin Zeidler said, “Repurposing MTX has the potential to provide a new, molecularly targeted treatment for MPN patients within a budget accessible to healthcare systems throughout the world – a development that may ultimately provide substantial clinical and health economic benefits.”

The research paper 'Low-dose methotrexate in myeloproliferative neoplasm models' was published in Haematologica, the journal of the European Hematology Association and the Ferrata Storti Foundation.

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