*Update on this study at the bottom of this article
We have all benefited from past research – and taking part in current studies will help the NHS improve patient care and treatment in the future.
“Other people have tested the drugs we’re taking now, so I felt I should help the research that could bring new treatments – it’s really a moral issue,” said Janet Hannah, as she became one of the first people in the world to take part in a clinical trial for a new treatment for a rare inflammatory muscle disease as part of a research study at Salford Royal.
Immune-mediating necrotizing myopathy (IMNM) is a type of myositis where muscle damage causes extreme weakness and fatigue, which can lead to difficulties in everyday life such as problems with walking, standing, or lifting.
In IMNM, your immune system—which usually protects you from infections by attacking problem cells—mistakenly attacks your muscles through a system called the complement pathway. This pathway destroys or kills muscle cells which results in the symptoms of IMNM.
It doesn’t yet have a cure, although there are treatments to help manage its debilitating symptoms. However, these treatments were not developed specifically for this disease, and they can have side effects.
Janet, a former outdoor instructor, fell runner and mountaineer, was diagnosed with the condition five years ago and says it’s had a devastating effect.
The treatment Janet volunteered to test as part of a randomized controlled trial is designed to target the cause of IMNM, stopping the complement process before it continues to kill muscle cells. The study involved daily injections under the skin of an investigational drug, zilucoplan, or placebo, for eight weeks, given by the patients themselves at home.
Janet, from Saddleworth, added: “This drug is trying to specifically target the disease, rather than being repurposed from something else. I was nervous about trying a new treatment but I was able to continue my other treatment alongside it.
“This isn’t just about me, I really hope other people might benefit from the research that’s being done.”
Professor Hector Chinoy (Principal Investigator) at Salford Royal said: “IMNM affects about 21 people in a million so it is very rare but it has very severe, debilitating effects and current treatment options are limited.”
Dr James Lilleker (Sub Investigator) added: “It’s important that we carry out research into more targeted treatments and we’re delighted that Janet agreed to take part in this important study.”
*Update March 2023: Sadly in this instance, the study didn’t find a beneficial effect but our team are grateful to all those who took part and are continuing their efforts to identify ways to help those affected. The study has now been reported in The Lancet Rheumatology, a leading medical journal.
Dr Lilleker added: “Sharing negative results is crucial for advancing scientific research and improving our understanding of diseases. This clinical trial has helped us to learn more about the cause of immune mediated necrotising myopathy and the role of the complement system. As a consequence, we can instead focus our efforts on other promising treatments.”