Scientists achieve breakthrough in the treatment of ‘neglected’ polymyalgia rheumatica
Scientists have had success treating a ‘neglected’ inflammatory disease, polymyalgia rheumatica, with a drug that could offer patients an alternative to steroids.
The study, conducted by Anglia Ruskin University (ARU) and published in the New England Journal of Medicine, describes a successful trial of sarilumab. The drug, approved in Britain to treat rheumatoid arthritis, blocked the protein interleukin-6, which can cause inflammation.
Polymyalgia rheumatica (PMR) is characterized by pain and morning stiffness in the shoulder and hips and affects people over the age of 50. It can significantly affect quality of life and is currently mainly treated with the steroids glucocorticoids.
Although glucocorticoids can control the condition, more than half of PMR patients experience a relapse of their condition when they reduce their steroid medication. Interleukin-6 has been implicated in the pathophysiology of PMR because circulating elevated levels and increased tissue expression of interleukin-6 have been found in PMR patients.
During the year-long clinical trial conducted by researchers, 118 patients received either twice-monthly injections of sarilumab or a placebo. The sarilumab group received a tapering dose of glucocorticoid for 14 weeks in combination with bimonthly injections of sarilumab, while the placebo group received a tapering dose of glucocorticoid for 52 weeks.
The primary outcome at the end of the study was sustained remission of the condition. This happened in 28% of people taking sarilumab, compared to 10% of people taking the placebo. After achieving remission at 12 weeks, there were more disease flares in the placebo group (57%) compared to those who received sarilumab (24%).
Lead PMR expert and senior author of the study, Professor Bhaskar Dasgupta, from the Medical Technology Research Center at Anglia Ruskin University (ARU), said: “Polymyalgia rheumatica is a poorly managed and neglected condition for which current treatment is unsatisfactory and can have long-term side effects. Patients can relapse while tapering their medications, and these relapses currently have very limited treatment options.
“Our findings show promise that sarilumab can be used to treat PMR and improve outcomes for people coming off steroid medications.
“This is an exciting development that has the potential to improve treatment options for a condition common in older people. PMR is the most common reason for long-term steroid prescriptions. Any effective drug that can spare the use of steroids would be a should have a major impact.” on reducing the serious side effects of such steroids, including diabetes, osteoporotic fractures and infections.”
The research was funded by Sanofi and Regeneron Pharmaceuticals.
Earlier this year, a review paper was published in the journal Nature reviews by Professor Dasgupta and colleagues highlighted the emerging view that relapsed PMR patients also have underlying giant cell arteritis, in which the main blood vessel aorta and its branches become inflamed. Researchers suggested that the two should be treated as linked conditions under the term GCA-PMR Spectrum Disease (GPSD).
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Magazine reference:
Tomelleri, A., et al. (2023). Disease stratification in GCA and PMR: state of the art and future perspectives. Nature Reviews Rheumatology. doi.org/10.1038/s41584-023-00976-8.
