Breakthrough finds arthritis drug could treat blood cancer

London: Blood cancer sufferers could be treated with a simple arthritis drug, according to scientists at a leading British university.

Martin Zeidler, from the University of Sheffield's Department of Biomedical Science, and his colleagues have found 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 the disease.

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

PV is a type of blood cancer which causes an overproduction of red blood cells.

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.

"We have now shown pretty conclusively that we can use this approach to treat mouse models of human MPNs, results which provide a much more tangible prospect of success in humans," he 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."

Every year 3,000 people in the UK are diagnosed with Polycythemia Vera. Patients suffer from itching, headaches, weight loss, fatigue and night sweats.

Current treatments do not slow the disease progression and provide little relief from symptoms, the University said in a release today.

The team now hope to go on to a full clinical trial early next year.

The research paper was published in Haematologica, the journal of the European Hematology Association and the Ferrata Storti Foundation.