Researchers at the University of Queensland (UQ) have developed a breakthrough vaccine-style rheumatoid arthritis treatment targeting the underlying cause of the disease rather than the symptoms.
Lead researcher Professor Ranjeny Thomas from UQ Diamantina Institute said the treatment worked by teaching the patient’s immune system to ignore a peptide it wrongly deemed to be a foreign enemy.
“It’s rather re-educating the immune system so that instead of responding to that enemy to actually stop responding or to make a counter regulatory peace-making effort against the enemy so that things will quieten down,” she said.
Researchers designed the treatment for people with the most common form of rheumatoid arthritis called CCP-positive.
In the first phase human trials they took a sample of patients’ blood, added an anti-inflammatory and the “foreign” peptide, then re-injected the modified cells back into the patient.
Professor Thomas said inflammation was noticeably reduced.
The results showing the treatment to be safe and effective have been published today in the influential journal Science Translational Medicine.
“It’s significant because it really is a new way of thinking about how to treat diseases like rheumatoid arthritis and it will have applications for other diseases as well which is similar like type 1 diabetes, multiple sclerosis.”
Researchers working on nano-particle vaccine
Professor Thomas said researchers were developing a ready-made vaccine to inject directly into patients.
“The way that we’ve published it in the trial is not something that is necessarily easy to take forward because it involves taking cells out, modifying them, putting them back,” she said.
“So we’ve also been working on a technology that involves doing this in a more practical way for patients and a more cost-effective way.
“This involves a nano-particle — small particle that encapsulates the peptide … and an anti-inflammatory drug in order to target the cells that we had taken out in the trial.
“But in this case we target them in the body directly. So it would be an injected product that targets those cells in the patient.”
Professor Thomas said she expected to start phase one clinical trials of the direct vaccine approach next year.
Rheumatoid arthritis affects about 500,000 Australians.
Erin Stafford, 29, from Nundah in Brisbane, was diagnosed with it when she was 18.
“When I was first diagnosed, everything like brushing my hair and putting on a shirt was extremely difficult,” she said.
“My mum used to have to help me to get to the toilet. Opening simple things like jars or a bottle of milk in the morning can bring you to tears.
It would be fantastic in the future if we could have a therapy that would be very effective very early in the disease.
Professor Ranjeny Thomas, lead researcher
“Everyday there’s something that’s a bit of a struggle … It’s very frustrating.”
Ms Stafford, who is completing her masters in psychology as well as working part-time, said she had to take four medications including an injection.
Her wrist joints have fused.
“Joints have a tendency to do that once they’ve been swollen for a while,” she said.
“So I don’t have any movement in my wrists anymore. Besides that mostly my knuckles and hands [hurt] and sometimes my foot as well.”
“I’ll one day need reconstructive surgery. Especially getting it at 18 years old, it’s kind of a given.”
While she was not involved in the human trials she said the early results were exciting.
“It’s amazing to hear that, just to know that someone’s still out there working on something. That we haven’t really been forgotten.”
Professor Thomas said if all went well a viable vaccine could be available within a decade.
“It would be fantastic in the future if we could have a therapy that would be very effective very early in the disease, and potentially even be used before the disease is going to hit in people that we can detect prior to the onset with good screening tools.”
Professor Thomas has been working on the treatment since 2003 with support from Arthritis Queensland.