Plans For A New Clinical Trial Offers Hope To Those With Motor Neurone Disease As A Result Of Research At Lancaster University
WINDERMERE, England, July 8, 2015 /PRNewswire/ --
A remarkable research initiative is emerging in the North-West of England through collaboration between the Association for the Independence of Disabled People (AID) and medical researchers at Lancaster University.
AID, founded by Mr. Paul and the Hon. Mrs. Ruth Adorian of Windermere, after Ruth contracted motor neurone disease (MND) aims to play a major role in raising the funds needed to finance this potentially exciting trial and has already raised over £80,000 before launching this appeal for public support.
MND is a neurodegenerative disease (other well-known examples are Alzheimer's and Parkinson's diseases). These diseases are all caused by similar neurodegenerative (nerve-damaging) processes that lead to cell death. If they take place in one part of the brain they lead to Alzheimer's disease, if in another part to Parkinson's disease, and if motor neurones are affected they lead to Motor Neurone Disease. This means that if a new treatment can be found for any of these diseases, it can probably be adapted to help treat people with the other diseases. It is currently estimated that there are 816,000 people with Alzheimer's disease and 127,000 with Parkinson's disease in the UK alone. Close to one million people of a total UK population of 64.1 million suffer from neurodegenerative disease, and through them the devastating effects of these vicious diseases touch many more lives.
Because these diseases appear to have a close link to diabetes (Alzheimer's disease has been called "the diabetes of the brain"), our researchers are testing existing diabetes drugs to see whether they can also help people suffering from neurodegenerative disease. The early results show real promise. If an existing diabetes drug is proven to help people with MND, the extended process of safety testing involved in launching a new drug has already taken place. A new treatment for MND would therefore be available on a much faster timeframe than for a new drug, prolonging life expectancy and dramatically improving quality of life for people with MND.
Liraglutide - a promising new drug treatment
The first candidate tested was exendin-4, a treatment that has now been superseded. It achieved excellent results in laboratory models of MND, and has since been tested in humans - but for Parkinson's disease. In a small clinical trial of people with Parkinson's disease, exendin-4 demonstrated good effects in preserving motor activity and cognitive power in the patients. Crucially, twelve months later the protective effects had been maintained.
The focus has now shifted to a new, more powerful, candidate. Liraglutide (Victoza®), the current market-leading diabetes drug, is superior to exendin-4 and its effects last much longer in the body (it has a half-life of 14 hours compared with 3.5 hours for exendin-4).
Liraglutide appears to be highly effective at protecting neurones from damaging influences. Laboratory studies have shown that the drug reduces key symptoms of Alzheimer's and Parkinson's disease. Clinical trials are now starting that look at the effects of Liraglutide on people with Alzheimer's and with Parkinson's disease. However, we also need to trial the effect of Liraglutide on people with MND.
Next step: a clinical trial in MND
In the light of these extraordinarily promising results we propose Liraglutide as an ideal, and urgent, candidate for a clinical trial in patients with ALS (the most common form of MND).
We plan to run a multi-centre UK double-blind randomised controlled trial, the gold standard in clinical trial design, to ensure that the results of the trial are as reliable as possible.
The trial will be led by Dr. Hedley Emsley (consultant neurologist) and Professor Christian Hölscher (leading researcher in neurodegenerative disease at Lancaster University) with the MND Care and Research Centre at the Royal Preston Hospital serving as the lead clinical unit in close collaboration with other UK centres.
Getting started
With pharmaceutical companies (as reported in the media) reducing their funding for dementia research, the majority of UK clinical trials are funded by the Medical Research Council, but as its funds are limited, many excellent and promising trials are not funded.
We currently estimate the total cost of the trial to be £450,000. If we can raise the full amount from philanthropic sources, we can then begin the trial. Raising a smaller amount, for example, £100,000 to £150,000, will increase our chance of securing the remainder from the Medical Research Council, but introduce a delay of at least six months.
All those concerned with the research, fund raising and implementation of the trial have been hugely encouraged by a decision taken by the Motor Neurone Disease Association, which has longstanding experience of participation in drug trials, to provide advice and organisational support for the trial.
Paul Adorian, Managing Director of AID said: "Once we have raised the necessary funds, the researchers can move forward with their plans to set up the trial.
"AID has already raised more than £80,000 in the county of Cumbria alone and we are confident that over a wider geographical area the full amount can be found for this innovative research."
AID welcomes donations for this worthy cause which can be accepted by credit/debit cards online at http://www.disabledday.org; by internet bank transfer payable to AID, Sort Code 20-45-28, Account No. 53690385 or by cheque made payable to AID MND Trial Fund.
Motor neurone disease is a progressive degenerative disorder of motor neurones that leads to weakness of key muscles, including those responsible for gripping, walking, speaking, swallowing and breathing. In most cases, survival is only 3-5 years from the onset of symptoms. There is currently no cure or effective treatment.
BACKGROUND INFORMATION
Lancaster University
Lancaster University's Faculty of Health & Medicine is committed to pursuing world-class research that brings together biomedical, medical, and social science researchers to tackle key challenges of the 21st Century within health and medicine.
Ranked third in our field in the 2014 Research Assessment Framework on the 'intensity weighted' score, equal first in 'research environment' and in the top 10 for 'research power', our high quality research and our commitment to teaching and to working with the health and care sectors means that the Faculty is influencing practice and thinking across a broad spectrum.
Lancaster has achieved a high profile for its research into neurodegenerative diseases (including Alzheimer's, Parkinson's and Motor Neurone Diseases), which includes research into techniques for earlier diagnosis, developing new treatments, and repositioning existing drugs to treat these diseases.
Association for the Independence of Disabled People
AID was established in 2014, is registered with the Charity Commission (No. 118537) and its objectives relate to the relief of the needs of physically disabled people. The Association is already involved in negotiations with hotel owners and the owners of buildings where the public is admitted with a view to improving facilities for disabled people with particular reference to the provision of appropriate equipment in rest rooms and bathrooms. This area of activity presents huge problems to seriously disabled people travelling with carers, as all too often those facilities provided in the majority of hotels in the UK fall far short of the needs of such people.
AID has reacted to an approach from Lancaster University for assistance with fund raising for this new drug trial and as the Chairman and founder of AID, the Hon. Mrs Ruth Adorian, was diagnosed with motor neurone disease eight years ago, it was felt appropriate to become involved in the fund raising process as there are 5,000 people in the UK currently suffering from motor neurone disease. (The Hon. Mrs Ruth Adorian, now 82m is the youngest daughter of the late Lord Wakefield of Kendal, better known in rugby circles as "Wakers" who captained the England rugby union team in the 1920's.)
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A remarkable research initiative is emerging in the North-West of England through collaboration between the Association for the Independence of Disabled People (AID) and medical researchers at Lancaster University.
AID, founded by Mr. Paul and the Hon. Mrs. Ruth Adorian of Windermere, after Ruth contracted motor neurone disease (MND) aims to play a major role in raising the funds needed to finance this potentially exciting trial and has already raised over £80,000 before launching this appeal for public support.
MND is a neurodegenerative disease (other well-known examples are Alzheimer's and Parkinson's diseases). These diseases are all caused by similar neurodegenerative (nerve-damaging) processes that lead to cell death. If they take place in one part of the brain they lead to Alzheimer's disease, if in another part to Parkinson's disease, and if motor neurones are affected they lead to Motor Neurone Disease. This means that if a new treatment can be found for any of these diseases, it can probably be adapted to help treat people with the other diseases. It is currently estimated that there are 816,000 people with Alzheimer's disease and 127,000 with Parkinson's disease in the UK alone. Close to one million people of a total UK population of 64.1 million suffer from neurodegenerative disease, and through them the devastating effects of these vicious diseases touch many more lives.
Because these diseases appear to have a close link to diabetes (Alzheimer's disease has been called "the diabetes of the brain"), our researchers are testing existing diabetes drugs to see whether they can also help people suffering from neurodegenerative disease. The early results show real promise. If an existing diabetes drug is proven to help people with MND, the extended process of safety testing involved in launching a new drug has already taken place. A new treatment for MND would therefore be available on a much faster timeframe than for a new drug, prolonging life expectancy and dramatically improving quality of life for people with MND.
Liraglutide - a promising new drug treatment
The first candidate tested was exendin-4, a treatment that has now been superseded. It achieved excellent results in laboratory models of MND, and has since been tested in humans - but for Parkinson's disease. In a small clinical trial of people with Parkinson's disease, exendin-4 demonstrated good effects in preserving motor activity and cognitive power in the patients. Crucially, twelve months later the protective effects had been maintained.
The focus has now shifted to a new, more powerful, candidate. Liraglutide (Victoza®), the current market-leading diabetes drug, is superior to exendin-4 and its effects last much longer in the body (it has a half-life of 14 hours compared with 3.5 hours for exendin-4).
Liraglutide appears to be highly effective at protecting neurones from damaging influences. Laboratory studies have shown that the drug reduces key symptoms of Alzheimer's and Parkinson's disease. Clinical trials are now starting that look at the effects of Liraglutide on people with Alzheimer's and with Parkinson's disease. However, we also need to trial the effect of Liraglutide on people with MND.
Next step: a clinical trial in MND
In the light of these extraordinarily promising results we propose Liraglutide as an ideal, and urgent, candidate for a clinical trial in patients with ALS (the most common form of MND).
We plan to run a multi-centre UK double-blind randomised controlled trial, the gold standard in clinical trial design, to ensure that the results of the trial are as reliable as possible.
The trial will be led by Dr. Hedley Emsley (consultant neurologist) and Professor Christian Hölscher (leading researcher in neurodegenerative disease at Lancaster University) with the MND Care and Research Centre at the Royal Preston Hospital serving as the lead clinical unit in close collaboration with other UK centres.
Getting started
With pharmaceutical companies (as reported in the media) reducing their funding for dementia research, the majority of UK clinical trials are funded by the Medical Research Council, but as its funds are limited, many excellent and promising trials are not funded.
We currently estimate the total cost of the trial to be £450,000. If we can raise the full amount from philanthropic sources, we can then begin the trial. Raising a smaller amount, for example, £100,000 to £150,000, will increase our chance of securing the remainder from the Medical Research Council, but introduce a delay of at least six months.
All those concerned with the research, fund raising and implementation of the trial have been hugely encouraged by a decision taken by the Motor Neurone Disease Association, which has longstanding experience of participation in drug trials, to provide advice and organisational support for the trial.
Paul Adorian, Managing Director of AID said: "Once we have raised the necessary funds, the researchers can move forward with their plans to set up the trial.
"AID has already raised more than £80,000 in the county of Cumbria alone and we are confident that over a wider geographical area the full amount can be found for this innovative research."
AID welcomes donations for this worthy cause which can be accepted by credit/debit cards online at http://www.disabledday.org; by internet bank transfer payable to AID, Sort Code 20-45-28, Account No. 53690385 or by cheque made payable to AID MND Trial Fund.
Motor neurone disease is a progressive degenerative disorder of motor neurones that leads to weakness of key muscles, including those responsible for gripping, walking, speaking, swallowing and breathing. In most cases, survival is only 3-5 years from the onset of symptoms. There is currently no cure or effective treatment.
BACKGROUND INFORMATION
Lancaster University
Lancaster University's Faculty of Health & Medicine is committed to pursuing world-class research that brings together biomedical, medical, and social science researchers to tackle key challenges of the 21st Century within health and medicine.
Ranked third in our field in the 2014 Research Assessment Framework on the 'intensity weighted' score, equal first in 'research environment' and in the top 10 for 'research power', our high quality research and our commitment to teaching and to working with the health and care sectors means that the Faculty is influencing practice and thinking across a broad spectrum.
Lancaster has achieved a high profile for its research into neurodegenerative diseases (including Alzheimer's, Parkinson's and Motor Neurone Diseases), which includes research into techniques for earlier diagnosis, developing new treatments, and repositioning existing drugs to treat these diseases.
Association for the Independence of Disabled People
AID was established in 2014, is registered with the Charity Commission (No. 118537) and its objectives relate to the relief of the needs of physically disabled people. The Association is already involved in negotiations with hotel owners and the owners of buildings where the public is admitted with a view to improving facilities for disabled people with particular reference to the provision of appropriate equipment in rest rooms and bathrooms. This area of activity presents huge problems to seriously disabled people travelling with carers, as all too often those facilities provided in the majority of hotels in the UK fall far short of the needs of such people.
AID has reacted to an approach from Lancaster University for assistance with fund raising for this new drug trial and as the Chairman and founder of AID, the Hon. Mrs Ruth Adorian, was diagnosed with motor neurone disease eight years ago, it was felt appropriate to become involved in the fund raising process as there are 5,000 people in the UK currently suffering from motor neurone disease. (The Hon. Mrs Ruth Adorian, now 82m is the youngest daughter of the late Lord Wakefield of Kendal, better known in rugby circles as "Wakers" who captained the England rugby union team in the 1920's.)
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