Behind the Headlines: Can 'miracle' therapy reverse MS?

A combination treatment shows promise for the treatment of severe early MS. Rachel Liddle reports.

What is the story?

A 'miracle' treatment for MS could allow sufferers to lead a normal life, according to media reports.

They reported that a combination therapy developed by British scientists reduced the rate of attack of the disease by up to 90 per cent.

The trial involving 27 MS patients showed that after treatment those confined to wheelchairs could walk and those who were blind could see, the newspapers claimed.

What is the research?

The two drugs used in the study were mitoxantrone, which is normally used to treat cancer, and glatiramer acetate (GA), an MS anti-relapse drug.

Both drugs have previously shown some effectiveness in treating MS. Mitoxantrone, however, is normally reserved for second-line therapy due to concerns over its cardiotoxic effects. A person is limited to 140mg/m2 a lifetime.

Researchers tested the effects of giving a sequential regimen of both drugs to 27 MS patients with very active relapsing-remitting disease.

They gave mitoxantrone to patients who had had MS for five or fewer years at a mean total dose of 74mg/m2 for the first four patients, 54mg/m2 for the next six patients and 48mg/m2 for the remainder. Once patients had been clinically stable for six months, they were given GA at a dose of 20mg daily for two months. After this point, mitoxantrone was withdrawn.

Two years before treatment began, patients had an average annualised relapse rate of 2.7. This fell to 0.106 over an average of three years of follow-up. This is equivalent to a greater than 90 per cent reduced relapse rate.

MRI scans of nine of the first 10 patients also showed a decrease in the number and volume of brain lesions following treatment.

The researchers reported no unanticipated side-effects of the combination treatment. But one patient did develop therapy-related leukaemia nine months after completing mitoxantrone therapy.

What do the researchers say?

Lead researcher Dr Mike Boggild, consultant neurologist at the Walton Centre for Neurology and Neurosurgery in Liverpool, said: 'If you have someone with high-risk disease, you have to treat them aggressively.'

He said standard treatments could reduce relapse rates by around 30 per cent, but were not effective enough in some patients with severe disease.

He said the centre had so far treated around 60 patients with the mitoxantrone and GA combination method.

'Five or six years on from initial treatment, they still haven't relapsed,' he said.

He admitted that further research data was needed to confirm the benefits of the mitoxantrone-GA combination.

But he added: 'I'm pretty confident that this is real, these patients don't do this in normal life, they develop disability very quickly.'

However, he conceded that doctors and patients would have to be sure that that the risks of the therapy did not outweigh the risks of the disease before opting for such an aggressive treatment regimen. A randomised controlled trial of the combination treatment is now under way at 10 neurology centres in England.

Patients with early disease who have suffered at least two relapses in the past two years will be eligible if they have additional signs of poor prognosis.

'What we're looking at is an approach to therapy with early active relapsing MS,' said Dr Boggild. 'The aim is to prevent disease, not reverse it.'

What is the expert opinion?

Dr Gavin Giovannoni, a consultant neurologist at the Institute of Neurology in London, said: 'It's a good scientific rationale from an immunological point of view.'

Mitoxantrone depletes levels of the t-cells that promote relapse, he explained. Immature t-cells that subsequently re-grow can be tackled more easily than mature t-cells by GA.

'This may be a way of making GA work quicker,' he said.

But he added that mitoxantrone alone had been shown to reduce relapse rate by 80-90 per cent, with effects lasting 12 to 18 months, so it was unclear whether the combination was boosting effectiveness further.

He said the current 10-centre trial being conducted wasn't powerful enough to answer that question.

'This is preliminary data and the results look exciting. Unfortunately, we don't have enough data on how safe it is,' he said.

Mitoxantrone is reserved as a second-line therapy for MS patients. Dr Giovannoni said the experimental regimen should only be given to patients in clinical trials until more safety data was available.


  • Preliminary data suggests that two drugs already used in the treatment of MS could reduce relapse rates more effectively if used together.
  • The treatment is still experimental. It does appear dramatically to reduce relapse rates in severe relapsing- remitting MS, but it could have serious side-effects.
  • One of the drugs, mitoxantrone, is known to carry serious risks, including cardiotoxicity and leukaemia.
  • A randomised controlled trial into the regimen is currently being conducted at 10 centres in the UK.
  • Patients with early MS, who have suffered at least two relapses in the past two years, may be eligible.


"MS wonder drug that can help the blind see and the disabled walk" - Daily Express

"The MS 'miracle'" - Mirror

"Double drug is 'a cure for MS'" - The Sun.

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