A common arthritis drug may also be an effective way to help treat patients with blood cancers—at one thousandth the cost of another drug that works the same way.
Myeloproliferative neoplasms (MPN) cause an overproduction of blood cells resulting in symptoms that include night sweats, itching, and tiredness.
MPNs are most often diagnosed in people in their 50s and 60s. Current treatment is limited to aspirin, removal of excess blood, and mild chemotherapy.
Recently, the drug Ruxolitinib has been developed and has been shown to provide relief, but costs more than £40,000 ($62,370) per patient per year and has yet to be approved by the National Institute for Health and Care Excellence (NICE).
A new study, published in PLOS ONE, shows Methotrexate (MTX) can work in the same way, says Martin Zeidler of the biomedical science department at University of Sheffield.
“Given that a year’s course of low-dose MTX costs around £30 [$47], the potential to repurpose MTX could provide thousands of patients with a much needed treatment option and also generate substantial savings for health care systems.
“Because MTX is a World Health Organization ‘essential medicine,’ this also means that this well understood drug could be used throughout the developing world.”
For the study, scientists used cells from the fruit fly Drosophila to screen for small molecules that suppress the signaling 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.
Few side effects
MTX is commonly used at low doses to treat inflammatory diseases including rheumatoid arthritis, Crohn’s disease, and psoriasis and 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 chemotherapies.
Working 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 for the treatment of 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 says.
“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,” says Nell Barrie, senior science information manager at Cancer Research UK.
“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.”
Cancer Research UK funded the work.
Source: University of Sheffield