Scientists have discovered that a common drug given to arthritis sufferers could also help to treat patients with blood cancers.
While ruxolitinib has been developed for patients with myeloproliferative neoplasms (MPN), scientists have discovered that methotrexate can work in the same way and at one thousandth of the cost.
Myeloproliferative neoplasms (MPN) cause an overproduction of blood cells. This creates a significant impact on quality of life, with symptoms such as night sweats, itching, and tiredness.
MPNs are most often diagnosed in people between the age of 50 and 70. The drug ruxolitinib has recently been developed, but it is costly. Otherwise, treatment is limited to aspirin, removal of excess blood, and mild chemotherapy.
Methotrexate was found to work in the same way as ruxolitinib by a research team from the University of Sheffield in the United Kingdom that was led by Martin Zeidler, DPhil, working together with colleagues from the Department of Haematology at the Royal Hallamshire Hospital, and funded by Cancer Research UK. The findings were published in PLOS ONE (2015; doi:10.1371/journal.pone.0130078).
“Given that a year’s course of low-dose MTX costs around £30 ($46), the potential to repurpose methotrexate could provide thousands of patients with a much needed treatment option and also generate substantial savings for health care systems,” said Zeidler.
“Because methotrexate is a World Health Organization ‘Essential Medicine’, this also means that this well-understood drug could be used throughout the developing world.”
In this study scientists used cells from the fruit fly Drosophila to screen for small molecules that suppress the signalling pathway central to the development of MPNs in humans.
Further testing confirmed this in human cells, even those carrying the mutated gene responsible for MPNs in patients.
Methotrexate is commonly used at low doses to treat inflammatory diseases that include rheumatoid arthritis, Crohn’s disease, and psoriasis. It has few side effects. It is also used in some cancers at much higher doses where the side effects are substantial and similar to other chemotherapy agents.
Working together with clinical colleagues at the Royal Hallamshire Hospital, Zeidler is now looking to undertake clinical trials to examine the possibility of repurposing low-dose MTX to treat MPNs.
“We have the potential to revolutionize the treatment of this group of chronic diseases: a breakthrough that may ultimately represent a new treatment option able to bring relief to both patients and health funders,” he added.
Nell Barrie, senior science information manager at Cancer Research UK, said: “Finding new uses for existing drugs is a great way to speed up improvements in treatment, as these drugs will have previously been through safety tests. Methotrexate is already used as a chemotherapy drug for several types of cancer, and this early research shows that at much lower doses it could have the potential to help treat certain blood disorders.”