Low-dose methotrexate has shown potential for treating symptoms of myeloproliferative neoplasms such as fatigue and night sweats.

A series of case studies published in the British Journal of Haematology in patients with myeloproliferative neoplasms taking the drug for other reasons showed unintended benefits of methotrexate in symptom management.

The researchers reported findings from 11 patients who happened to be taking methotrexate for other co-morbidities which showed statistically significantly lower symptom scores compared with historic controls.

One 56-year old man who had been taking methotrexate for rheumatoid arthritis began to complain of pruritus as soon as he stopped taking the drug. His haematocrit rapidly increased to 51% and he had to have venesection despite continuing hydroxycarbamide treatment, the UK researchers reported. Once methotrexate was started again his symptoms resolved.

The case series was done after previous reports by the same team showed low‐dose methotrexate to be a JAK/STAT pathway inhibitor and found it to be effective in mouse models of myeloproliferative neoplasms.

Study leader Dr Martin Zeidler from the University of Sheffield said the nature of evidence so far meant they could not draw firm conclusions but methotrexate showed real promise. The team  are keen to take the work further, with the hypothesis that adding methotrexate could be added to usual treatment to provide additional benefit.

“Despite the very small numbers, we still found significance which suggests this is quite a large effect,” he told the limbic in an interview.

“We have clinicians lined up to do this and a clinical trials centre in Birmingham who specialise in this sort of study and its all ready to go but what we don’t have the funding to do it.”

The team had received the go-ahead from blood cancer charity, Bloodwise, who had agreed to fund the research before hitting financial difficulties. A recent application to the Wellcome Trust had been rejected.

“The challenge now is to either relicense methotrexate for use in myeloproliferative neoplasms or build a sufficient evidence base that doctors are happy to use it off label,” said Dr Zeidler.

“We do now have the opportunity to apply for another round of funding with Bloodwise so we are hoping to resubmit but it is a very slow process.”