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A simple anti-arthritis drug could be an effective and inexpensive solution for treating patients with blood cancers, such as viral polycythemia (PV) and essential thrombocythaemia (ET), according to a landmark study of the disease. 39, University of Sheffield.
Under the direction of Dr. Martin Zeidler, Department of Biomedical Sciences of the University of Sheffield, and Dr. Sebastian Francis of the Department of Hematology of the Royal Hallamshire Hospital, as well as Oxford and Oxford Universities. Cambridge, the study results show that Methotrexate (MTX) – a drug on the list of essential drugs commonly used by the World Health Organization to treat rheumatoid arthritis – dramatically reduces the symptoms associated with disease.
Each year, about 6000 people in the UK are diagnosed with PV or ET, blood cancers associated with an overproduction of red blood cells (WBCs) or blood clotting platelets (ETs). Patients often suffer from itching, headaches, weight loss, fatigue and night sweats.
Although current treatments can usually control the increase in the number of blood cells, they do not relieve the sometimes debilitating symptoms that often have a significant impact on the quality of life.
Building on previous work funded by the Medical Research Council in the Zeidler Laboratory that identified methotrexate as an inhibitor of the JAK / STAT signaling pathway, this study examined hospital records to identify ET and PV patients already treated with methotrexate for other diseases.
Despite the small number of people involved and the presence of rheumatoid arthritis, these patients had significantly lower symptom scores than those who did not take methotrexate.
Deregulation of the JAK / STAT signaling pathway in humans is at the heart of the development of myeloproliferative neoplasms (MPNs), a collective term for evolving blood cancers such as ET and PV, as well as many inflammatory processes such as those associated with rheumatoid arthritis.
Laboratory results showed that low doses of methotrexate acted as a potent suppressor of activation of the JAK / STAT pathway – even in cells carrying the mutated gene responsible for MPNs in patients.
Dr. Zeidler said, "Although we still need to undertake a clinical trial to validate these findings, our results are very encouraging and suggest that a single drug has been used for nearly 40 years to treat the disease. Arthritis can bring significant relief to people with blood cancer.
"The patients we tested showed marked improvement in their symptoms, which conventional treatments were unable to provide.
"Given the very low cost of MTX, this research could provide effective therapy on a budget accessible to health systems around the world, which would provide a potentially significant economic and clinical benefit to health."
An 81-year-old PV patient based in California commented: "Methotrexate seemed to be doing a very good job in controlling itching and night sweats." My subjective MTX experience was PV vacation. "
MTX has been used for 40 years to treat inflammatory diseases such as rheumatoid arthritis, Crohn's disease and psoriasis. Although the mechanisms by which MTX acts in these diseases have not yet been understood, its safety and efficacy are well documented and millions of patients take it regularly.
Diseases such as rheumatoid arthritis are all characterized by inflammatory processes induced by JAK / STAT activity and the effectiveness of MTX in these inflammatory diseases could well be a consequence of its ability to attenuate the JAK / pathway. STAT.
The results of the study were published today (17 September 2019) in the British Journal of Hematology.
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