Motor neuron sufferers to test daily lithium pill treatment in clinical trial
David Rose
Hundreds of motor neuron disease sufferers are to be given a daily lithium pill in a clinical trial of a possible treatment for the devastating condition.

Scientists will test whether lithium carbonate, a cheap drug already prescribed for depressive illnesses, can slow the deterioration of patients who often survive little more than a year after diagnosis.

There is evidence that lithium can help sufferers, and up to 10 per cent of patients are thought to be taking the drug already. Scientists leading the £1 million, 18-month study say that this is ill-advised, because the drug can have serious side-effects ranging from aggravated skin conditions to an under-active thyroid, tremors, seizures and disorientation.

At present there is no effective treatment for the progressive illness, which damages the nervous system, leaving muscles wasted. Most patients are confined to a wheelchair and die within two to five years. About 50 per cent die within 14 months of diagnosis; only one patient in ten survives ten years. Very rare examples, such as Professor Stephen Hawking, live longer.


The trial, funded by the MND Association and the Department of Health, will begin in the first half of next year and aims to recruit 220 patients at ten MND care centres across England.

Professor Nigel Leigh, director of the centre at King’s College London, and one of the study’s leaders, said: “We are looking for something like a 20 per cent reduction in death rate at the end of 18 months, which is a huge effect.”

That would translate to an extra three to six months of life, or maybe more if treatment continued beyond the end of the trial.

About 5,000 people in Britain are living with MND, which can strike any adult at any time. Most patients were expected to live between 14 and 15 months after diagnosis, and the present treatment, riluzole, extended this only by about two months, Professor Leigh said.

Professor Hawking, 66, Lucasian Professor of Mathematics at Cambridge University and one of the world’s leading cosmologists, is an exceptional case. He discovered that he had amyotrophic lateral sclerosis — the most common form of MND — more than 40 years ago.

The way his body has defied the disease disqualified him from taking part in the lithium trial, Professor Leigh said. All those involved had to have the “normal” rapidly progressing disease if the study were to produce meaningful results.

Lithium is already licensed, mainly as a treatment for bipolar disorder, and costs 2.4p per 200-milligram tablet, suggesting that it would be offered to most MND patients if the trial showed that it had beneficial effects. The British trial follows a small Italian study that in February showed highly promising results from treating MND patients with lithium. But doctors are treating this research with caution because it did not involve the use of a placebo.

A larger trial in America looking at the effects of lithium on everyday function rather than survival is also due to start in the next few months.

Laboratory experiments suggest that lithium can protect nerves and may even promote new nerve growth.

Brian Dickie, director of research and development at the MND Association, said: “Any drug that can be shown to slow the course of MND in a significant way and is safe and well tolerated will be an important advance for people living with this disease.”

Professor Leigh strongly advised MND patients against taking lithium until more was known about its effects.

“It’s tempting, but please bear with us,” he said. “It’s much safer to wait because it may turn out that this is not good for patients. We don’t advise patients to take it by private arrangement with their GP.”



Case study: I'm lucky I can still speak

May 8, 2006, is memorable for Mark Carr not only as his 44th birthday but also as the day on which he was told he was suffering from the ravages of motor neuron disease (MND), a terminal illness.

He said: “I had been showing alarming symptoms for about 18 months. I'd been shaking, dropping things, had no strength in my arms, but no one could tell me what was wrong. MND is hard to diagnose and it was a case of ruling everything else out. I was finally told it could be MND or HIV, and the doctor said, 'If it's HIV, I can treat you.' It wasn't.”

Mr Carr, a former drug testing consultant from Wakefield, West Yorkshire, is now confined to home and has difficulty moving his arms, neck and upper body. He is reliant on a £6,000 ventilator machine to breathe.

“I used to play football, used to do a lot of DIY, and now I'm having to sell my house because I can't get downstairs,” he said. “The machine is keeping me alive, because my lung muscles do not work any more.

“People with MND deteriorate in different ways. I get about ten visits a week from medical and social services, and I try to talk to others who are in a similar position. I suppose I'm lucky that I can still speak and swallow for myself, drink wine and eat. You have to try and make the most of your time left.”

Mr Carr said that he had not asked for lithium carbonate because of concerns over the side-effects, and was unsure whether he would be eligible for the latest trial. “But it's clearly an excellent step in the right direction,” he said. “At present the only drug available is riluzole, which can give you an extra three months. You think, thanks very much, but it's not really enough.

“The trial may lead to disappointment, but any new drug is certainly worth checking out. It's one step of many that may be required on the journey to finding an effective treatment for MND.”



http://www.timesonline.co.uk:80/tol/...cle5068728.ece