Scientists at the University of Sheffield and Royal Hallamshire Hospital in the UK have found that an existing arthritis drug—methotrexate—might also come to the aid of patients with blood cancers.
Arthritis Drug Could Treat Blood Cancer
1 min read Premium comments
Secondary
According to the July 7, 2015 news release, treatment for myeloproliferative neoplasms (MPN) is limited to aspirin, removal of excess blood and mild chemotherapy. The UK team found that methotrexate (MTX) might work well in treating these patients.
Dr. Martin Zeidler of the biomedical science department at the University of Sheffield said in the news release, “Given that a year’s course of low-dose MTX costs around £30 [about $46.90 USD], 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 Organisation ‘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.”
Dr. Zeidler told OTW, “I think there are two things that surprised me about this finding. Firstly that the mechanisms of action of methotrexate in rheumatoid arthritis wasn’t known and that our finding could explain how this drug works and second, that nobody else had found it before.”
“We are currently in the process of undertaking further patient and mouse in vivo experiments. We are also preparing an application to launch Phase II clinical trials in polycythemia vera patients to examine the possibility of repurposing MTX for the treatment of MPNs. It will probably be another 12-18months before this trial starts. However, given that MTX is so well understood in RA patients there is nothing to stop haematologists prescribing MTX off-label right now [not that we are recommending this approach!]. As such it COULD be available right now…but undertaking proper Phase II and III trials would be required before it could be formally repurposed.”
React:
Discussion
This is a fascinating development. In my practice we've seen similar outcomes with the revised protocol. The key differentiator seems to be patient selection criteria. Has anyone else noticed the correlation with BMI thresholds?
Great point. I'd push back slightly on the conclusion, the sample size in the cited study is too small to draw population-level inferences. That said, the directional signal is compelling and worth a larger RCT.
We implemented a similar approach last year. Early results are promising but we're still gathering 12-month follow-up data. Happy to share our protocol if anyone is interested.
Join the conversation
Orthopedic professionals are discussing this. Sign in and upgrade to read every comment and add your voice.