The arthritis drug Toclizumab could be a boon for hundreds of children suffering from severe arthritis. It has been re-launched in children after being already licensed for the treatment of adults. At least two thirds of children taking tocizilumab have been able to return to a normal life, after many were bedridden or forced into wheelchairs by the disease, the clinical studies reveal.
Doctors claim the drug has transformed the outlook for victims of systemic juvenile arthritis, a severe inflammatory disease that can affect children as young as 18 months. Around 2,500 children in Britain are currently living with the disease, which can persist into adulthood and cause significant disability. Till date the therapy included anti-inflammatory drugs such as corticosteroids which may cause severe side effects and often do not slow progression of the disease.
The latest clinical study of 112 children showed that after just three months of treatment with tocizilumab nearly three-quarters had a 70 per cent improvement in their condition, compared with eight per cent taking a placebo, or dummy drug. After a year two-thirds of children had a 90 per cent improvement in their symptoms.
Dr Eileen Baildam, consultant pediatric rheumatologist Alder Hey Children’s Hospital, Liverpool, has treated 12 children with the drug and seen them all make a remarkable recovery. She said, “These are very sick children, the disease affects every single joint in their bodies as well as heart and lungs. They can die from heart failure and other conditions if they don’t get treated. It’s much worse than rheumatoid arthritis in adults. To control flare ups we’ve had to use huge doses of steroids which have bad side effects or another drug called methotrexate, and they tend to wear off.”
Dr Baildam said the new drug, already prescribed for adults with rheumatoid arthritis where it is not controlled by other medication, is given by intravenous infusion once a month. “The trial results show two-thirds get a 90 per cent response, which is almost complete recovery and with few side effects. But in practice every single one of my patients has gone back to a normal life, even if they have had the disease for some time. They have been able to get out of their wheelchairs, it’s fantastic,” she said.
Toclizumab is a laboratory-manufactured antibody that blocks the activity of interleukin 6 (IL-6), an important immune system signaling molecule that underpins many inflammatory processes. It may be given either on its own or in combination with standard disease modifying anti-rheumatic drugs. The treatment made by Roche is being licensed from today for use in children aged two and older.
The £9,000 annual cost of tocilizumab, also known as RoActemra, is the same as other advanced ‘biologic’ drugs already used for arthritis, but the NHS rationing body will have to decide whether it offers value for money in children.
Dr Baildam said, “I hope and expect it will be approved and I think it should be given to children as soon as they are diagnosed to limit the disability caused by this dreadful disease.” Professor Patricia Woo, professor of pediatric rheumatology at London’s Great Ormond Street Hospital, which also took part in the trial, said, “Systemic juvenile arthritis can be a devastating disease. It strikes often very young children, causing chronic illness, pain and disability. It is hugely encouraging to have an effective medicine now available to alleviate symptoms, control disease activity and potentially hold back the worst long-term consequences of the disease.” Ailsa Bosworth, chief executive of the National Rheumatoid Arthritis Society (NRAS) said the treatment offered ‘a future with hope’ to affected children and their families. “It causes immense distress and tocilizumab provides both families and clinicians with a new and effective treatment option which is greatly welcomed,” he said.
Rheumatoid arthritis treatment and heart disease risk
Another study revealed that rheumatoid arthritis medication could cut cardiovascular risk in those with the condition. Research published in journal Arthritis Research & Therapy noted that individuals with rheumatoid arthritis are at an elevated risk of dying due to cardiovascular disease.
This was found to be due to disease-related inflammation on top of the risk factors already affecting the general population. Treatment with disease modifying antirheumatic drugs (DMARDs) was seen reduce the risk of heart disease among these patients.
It was also found that a new cardiovascular event such as heart disease, stroke or deep vein thrombosis could be predicted by the intensity of the arthritis as well as the presence of diabetes, high blood pressure and triglyceride levels.
Arthritis-style immunotherapy shows promise for heart failure