26 September 2019

A low-cost drug that has been used for 40 years to treat arthritis could be used for patients with myeloproliferative neoplasms (MPNs), according to a new study.

The research is a retrospective evaluation of 11 MPN patients, led by Dr Martin Zeidler from the University of Sheffield's Department of Biomedical Science and Dr Sebastian Francis from the Department of Haematology at the Royal Hallamshire Hospital.

Previous work by Dr Zeidler demonstrated that methotrexate is an inhibitor of the JAK/STAT signalling pathway, which is thought to be central to the development of MPNs such as polycythaemia vera (PV) and essential thrombocythemia (ET).

To investigate further, the team examined hospital records to identify existing ET and PV patients who were already taking methotrexate for other diseases, like rheumatoid arthritis and psoriasis.

In these cases, the team showed that methotrexate significantly reduced the symptoms associated with PV and ET. Despite the small number of patients involved and the presence of comorbidities, these patients reported significantly lower symptom scores, compared to a historical control cohort of patients not taking methotrexate.

The findings were published recently in the British Journal of Haematology.

Dr Zeidler said: “While we still need to undertake a clinical trial to validate these findings, our results are very encouraging and suggest that a simple drug that has been used for nearly 40 years to treat arthritis can provide significant relief to blood cancer sufferers.

“Patients we tested showed a pronounced improvement in symptoms, something conventional treatments have been unable to provide.

“Given the very low cost of methotrexate, this research could offer an effective therapy on a budget accessible to healthcare systems throughout the world - marking a potentially substantial clinical and health economic benefit.”

Source: Francis, S., Thomas, S., Luben, R., Sousos, N., Mead, A., Snowden, J.A., Zeidler, M.P. (2019) “Low-dose methotrexate: potential clinical impact on haematological and constitutional symptoms in myeloproliferative neoplasms”, British Journal of Haematology, doi: 10.1111/bjh.16193


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