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Cancer drug may slow progression of MND and offer patients first breakthrough therapy in decades (2017)

Author Zhangqi Views Posted at 2018/11/27

By Helen McArdle in HeraldScotland.com August, 7, 2017

PATIENTS in Scotland are being sought for the first clinical trial in 20 years into a potential new treatment for motor neurone disease that scientists hope will slow its progression.

Researchers will test a drug already prescribed for kidney cancer to find out if it is effective at boosting the body's immune system to stall the nerve damage associated with MND.

They hope to recruit at least 15 participants in Scotland who have Amyotrophic Lateral Sclerosis (ALS) - the most common form of MND - but the £1.5 million study will require a total of 216 MND patients across research centres across the UK and France.

It signals the first potential breakthrough in the fight against the deadly and incurable condition in decades.

Riluzole, the first drug shown prolong survival in MND patients, has been available since 1996 but the only other drug approved since - edaravone - has marginal effects, and is licensed in the US but not Europe.

However, scientists believe that interleukin-2 - already prescribed to kidney cancer patients - could offer a new hope.

Read more: Footballer six times more likely to develop MND - heading ball may be to blame

It has long been known that interleukin-2, a naturally-occurring protein which regulates the activity of white blood cells, boosts the number of so-called 'T-reg' cells - regulatory T cells - which are key to immune system function.

There is also evidence of a relationship between the concentration of T-reg cells in a person's bloodstream and their survival with MND.

It is thought they protect the motor neurone nerve cells which are ravaged by the disease, leading to muscle wastage, disability and eventually death.

The condition has been pushed into the spotlight by high-profile patients and campaigners including former Scotland rugby ace Doddie Weir, Rangers footballer Fernando Ricksen and the late political aide Gordon Aikman, who campaigned to improve MND services before his death in 2017.

To date interleukin-2 has been trialled in mice modelled to simulate MND, but researchers hope that it will show positive results in humans.

Professor Nigel Leigh, who is heading up the clinical trial, said: "The story goes that the more T-regs you have whizzing around your body and, we think, the brain, the better you do.

"That's a statistical correlation, so it didn't require genius on our part to say 'well, supposing we could boost these T-reg cells, would that be a good thing?'.

"We don't expect it to be a quick fix cure. It's disease modifying, and the question is - does it work at all? And how big is that effect if it does?"

Participants in the study, known as MIROCALS, will be given a low dose of interleukin-2 over five days once a month for 18 months.

Newly-diagnosed patients with no symptoms will be included, but all participants will have blood and spinal fluid analysed at the end of the trial to gauge the disease progression.

"We know for sure that their T-regs are going to be significantly increased," said Prof Leigh. "The question then is does it slow down the disease significantly?"

The Scottish arm of the study will be led in Glasgow, but will be open to eligible patients from across Scotland.

Dr. George Gorrie, consultant neurologist at Queen Elizabeth University Hospital, who will be leading the trial in Scotland, said: “I am hopeful this study will lead to further drug trials in MND across the country.

"Enabling patients in Scotland to take part in clinical research is key to advancing the development of effective treatments against this terrible disease.”

Lawrence Cowan, Chair of MND Scotland and best friend of Gordon Aikman, said it was "an historic moment" in the fight to find a cure.

He said: “Gordon and I always talked about bringing clinical trials here. I wish so much that he was still with us to see it happen.

“This is the start of something incredible - the new phase of Scotland’s MND research revolution. I want us to bring more trials here. That’s why MND Scotland has created a 'time for trials fund' which will invest over £1.5 million to help deliver our ambition of bringing more drug trials to Scotland.

“I believe we can be the generation to cure MND. Together we can make it happen.”

First Minister Nicola Sturgeon, who has supported MND research projects in the past, said she was delighted at the development.

She said: “Research and clinical trials are an essential part of work to develop effective new treatments for Motor Neurone Disease and the Scottish Government has recently provided significant support towards a range of research work.

“We want to ensure that people living with MND have access to the best possible care and support across the country. That is why I am pleased that patients in Scotland will now be able to access this trial which I hope can be a major step towards the goal of a cure for this devastating disease.”


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