A revolutionary gene therapy could offer new hope for the 32.5million Americans suffering from a crippling condition that makes it difficult to walk.
Osteoarthritis is caused by the gradual breakdown of the cartilage that cushions the joints, leading to pain, stiffness and impaired movement. It is difficult to treat, with patients often needing regular pain relief and joint replacement surgery.
But a first-in-human trial of a novel therapy suggests doctors could soon relieve the pain for at least a year with a single injection.
Scientists at the Mayo Clinic injected the knees of nine osteoarthritis patients with a genetically-altered benign virus that caused cells to make an anti-inflammation molecule.
Over 12 months, participants reported they had reduced pain and were better able to move their knee. There were no serious safety issues.
Dr Christopher Evans, a physical medicine expert who led the study, said: ‘This could revolutionize the treatment of osteoarthritis. This study provides a highly promising, novel way to attack the disease.’
This was only a Phase 1 trial, the initial stage of testing for a new medical treatment, so more tests are needed to solidify dosage, frequency and pricing.
But the early results were heralded as promising, with doctors now hoping to be able to get the drug to patients within the next few years.
The nine participants in the early trial had no serious reactions to the treatment (stock image)
Osteoarthritis is the most common form of arthritis, and is linked to wear and tear on joints caused by aging, repeated stress from a job or sport, obesity or previous injury.
It is most common in the hands, knees, hips and spine, and tends to be diagnosed at around 50 years old. Patients are normally female, and may be overweight or have a family history of the disease.
The repetitive stress on joints from overuse and injury can damage cartilage, while aging means cells are less able to repair the tissue, leading to the condition.
Doctors treat osteoarthritis with over-the-counter painkillers and lifestyle changes, and may also recommend a joint replacement in severe cases. Hyaluronic acid injections are also available, but these normally only last around six months.
Painkillers and injections only target the pain, while joint replacements are used to resolve the cause of the disease.
In osteoarthritis, joints affected by the condition typically contain high levels of interleukin-1 (IL-1), a molecule linked to high levels of inflammation, pain and cartilage loss.
Dr Evans and his team have focused on reducing levels of this molecule by targeting it with the molecule IL-1 receptor antagonist (IL-1Ra), which has been shown to reduce IL-1 levels.
In the study, published in Science Translational Medicine, scientists recruited participants who were suffering from osteoarthritis.
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Patients were then injected three times in one of the knees with osteoarthritis with an altered virus carrying the IL-1Ra gene.
The gene entered cells in the knee, and caused them to start to make the anti-inflammatory molecule.
Tests on blood and fluid found in the cavities of joints after the injection showed there were much lower levels of inflammation than before.
The study was only meant to test whether the treatment was safe to use in humans, but doctors said that participants also said the injections helped to ease their pain.
Only two minor safety events were reported, and both were effusions, or abnormal fluid accumulations, that increased pain but resolved with treatment. The treatment used was not specified.
It wasn’t clear how long pain relief from the injections may last. It also wasn’t clear whether it could be used to ease pain in other joints like the fingers.
Dr Evans added: ‘Any medications you inject into the affected joint will seep right back out in a few hours.
‘As far as I know, gene therapy is the only reasonable way to overcome this pharmacologic barrier, and it’s a huge barrier.’
In similar early studies, Dr Evans and his team added the IL-1RA gene into a harmless virus called AAV and tested it in the lab.
In these models, they found that the virus successfully infiltrated the cells that make up the linings of joints and cartilage and caused the molecule to be made.
In 2015, the team then received approval to start testing the drug in humans, but regulatory hurdles meant it took another four years before this could begin.
The scientists are now excited to offer their treatment to a wider group of participants.
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