A drug launched today could ease the agony of hundreds of children suffering from severe arthritis.
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.
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.
Until now, commonly used treatments have been anti-inflammatory drugs such as corticosteroids which may cause severe side effects and often do not slow progression of the disease.
But trial results of 112 children show 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 paediatric rheumatologist and triallist at 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.
The new drug is a laboratory-manufactured antibody that blocks the activity of interleukin 6 (IL-6), an important immune system signalling 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 paediatric 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.’