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A drug which has already been in use for decades to treat liver disease could be an effective treatment to slow down progression of Parkinson’s disease, scientists from the University of Sheffield have discovered.
The research led by academics from the Sheffield Institute for Translational Neuroscience (SITraN), in collaboration with scientists from the University of York, supports the fast-tracking of the drug ursodeoxycholic acid (UDCA) for a clinical trial in Parkinson’s patients.
Following the motto "EveryAugustUntilACure", SITraN PhD students Jodie Stephenson and Alejandro Lorente Pons organised a revival of last year's Ice Bucket Challenge for Motor Neurone Disease (MND), also known as Amyotrophic Lateral Sclerosis (ALS). For a small donation towards MND research, visitors to Endcliffe Park in Sheffield had a chance to drench the scientists and find out about the research into Motor Neuron Disease (MND) at SITraN, as well as the charity work of the MND Association South Yorkshire branch. The team raised more than £600 in total which will go towards MND research. The event was covered in a radio interview with Jodie, Alejandro and Ann Quinn from the MND Association South Yorkshire branch by sheffieldlive (from 7:00 –20:13 mins). For more photos visit the MND Association South Yorkshire Facebook Page. An article on the event can also be found on the MND Association website.
A pioneering trial investigating the safety and efficacy of diaphragm pacing used to alleviate breathing difficulties for people with motor neuron disease (MND), has revealed the intervention is not generally beneficial to patients.
A team of MND specialists, led by researchers from the Sheffield Institute of Translational Neuroscience (SITraN), conducted the first randomised controlled multi-centre clinical trialto assess the risks and benefits of the intervention for patients.
The diaphragm pacing device was approved for humanitarian use in MND patients in 2011 by the US Food and Drug Administration. Despite a lack of conclusive evidence for the benefits of the intervention, diaphragm pacing is now widely offered to patients with MND around the world. The device, which is similar to a heart pacemaker, sends electrical impulses to stimulate the main breathing muscles in the diaphragm.
The results of the DiPALS study, published in the journal Lancet Neurology, show that diaphragm pacing was not beneficial when used in addition to non-invasive ventilation (NIV) where slightly pressured air is delivered into the lungs through a face mask. In fact, patients who used diaphragm pacing lived on average 11 months shorter than those who used NIV alone.
Lead researcher, Dr Christopher McDermott from SITraN, who is based at the University of Sheffield, said:
“The results from the DiPALS study are incredibly disappointing, because as a researcher and an MND doctor you start out with some hope that this is a treatment that can be truly beneficial for people living with MND.
“Unfortunately, DiPALS did not show any benefits for diaphragm pacing in MND and, in fact, our study showed that it may actually be harmful. Although the results are disappointing, it was an important study to carry out as this evidence shows us that for most people there is no benefit in having diaphragm pacing and that the major surgery needed is something people living with MND should not go through.”
He added:“We carried out the DiPALS study because breathing difficulties are a major problem in MND, especially during the later stages of the disease. Current guidelines recommend non-invasive ventilation (NIV) should breathing difficulties arise, however we know that the benefits of NIV are limited and that NIV does not suit everyone. Therefore, research into complementary and alternative techniques to help with breathing is needed.
“We were aware of the work in the United States on diaphragm pacing in MND and we wanted to know if it would be beneficial for our patients. Therefore, we decided to design a randomised controlled clinical trial of diaphragm pacing in MND.
“Funding bodies like the NHS and NICE need this evidence of benefit before a treatment can be made available in the UK. Also, because it is a treatment that requires a major operation, we wanted to make sure beyond reasonable doubt that diaphragm pacing is worthwhile for patients, adding sufficient benefit such as living longer and a better quality of life.
“We established collaboration within the MND community, including the Dementia and Neurodegenerative Disease Research Network (DeNDRoN), and the MND Association and we then applied for funding from the National Institute for Health Research (NIHR)to carry out this clinical trial. By collaborating with other UK MND Care Centres we were able to carry out a well-designed clinical trial to determine if this intervention was beneficial to people living with MND.”
Dr McDermott concluded: “MND is an awful disease and affected individuals, loved ones and the health care professionals involved in providing care, are understandably always eager to consider new treatments. It is important that new treatments are evaluated in rigorous trials to demonstrate their benefit and importantly ensure no harm is done. The result from DiPALS demonstrates that the increasing “nothing to lose” approach is inappropriate and we should not lower our standards by starting treatments without clear evidence of benefit.”
“I am always humbled by the precious time and effort individuals give up to take part in our research studies. Those individuals who participated in DiPALS have contributed enormously to ensuring we understand the effects of diaphragm pacing in patients with MND and will ensure that we now put our focus and resources on developing other treatments that may help.”
This project was funded by the National Institute for Health Research Health Technology Assessment (NIHR-HTA) Programme (project number 09/55/33) and the Motor Neurone Disease Association of England, Wales and Northern Ireland.
DiPALS Study Group (2015) Lancet Neurology. http://www.thelancet.com/journals/laneur/article/PIIS1474-4422(15)00152-0/abstract
Podcast: Dr Chris McDemott discusses study with The Lancet’s Richard Lane http://www.thelancet.com/pb/assets/raw/Lancet/stories/audio/laneur/2015/laneur_300715.mp3
There is also a great blog published by the MND Association to explain the result further https://mndresearch.wordpress.com/
More information on related topics can be found on the MND Association website:
For more information on non-invasive ventilation in MND visit: www.mymnd.org.uk
On Friday 17th July 2015 SITraN opened its doors to the public for an afternoon of talks, tours and demonstrations. The yearly event gives visitors a behind-the-scenes look at the institute’s research into motor neuron disease (MND) and related disorders such as Parkinson’s and Alzheimer’s disease.
Visitors received a warm welcome by SITraN Director Prof Dame Pam Shaw and an update on last year’s highlights. Guest of Honour Sally Light, Chief Executive of the MND Association, presented the charity’s vision and work in partnership with SITraN praising the multidisciplinary, collaborative approach to developing new treatments for MND. Short talks by MND Association/Kenneth Snowman Lecturer Dr Richard Mead, Parkinson’s UK Fellow Dr Heather Mortiboys and Alzheimer’s Society Fellow Dr Claire Garwood followed. The speakers who all received prestigious charity awards for their research presented current strategies for therapy development in MND, Parkinson’s and Alzheimer’s focusing on the role of astrocytes, star-shaped brain cells that support nerve cells, and mitochondria, the cell’s energy generators, which show similar functional defects across neurodegenerative diseases.
Astrocytes (left), star-shaped brain cells that surround and support nerve cells, and mitochondria (right), the cell’s energy generators, play important roles in neurodegenerative diseases.
During the refreshment break visitors were treated to a selection of cakes and cookies home baked by SITraN staff and students.
Guests had a chance to chat with the researchers and browse the information stands provided by Parkinson’s UK, the Alzheimer’s Society and the South Yorkshire MND Association. SITraN PhD student Cassy Ashman presented her exhibition “MND: from Loss to Hope”, a joint project created with art therapist Cecilie Browne for the Sheffield Festival of the Mind to generate MND awareness. The research advisory groups for MND (http://smndrag.group.shef.ac.uk)and Dementia were also present with posters and information for anyone interested to join. The groups are hosted by SITraN and members are critically involved in many of the centre’s clinical research projects e.g. the “telehealth in MND” project (TiM), the customisable MND neck collar and the new “myNIV web resource for non-invasive ventilation in MND” (www.mymnd.org.uk).
A team of SITraN volunteers then took the visitors on a tour to have a look into the laboratories and visit work stations especially set up for the Open Day to discover more about the research taking place at SITraN. Tours were organised around three themes including “Gene Expression”, “Drug Discovery” and “Clinical Research” ranging from basic science to pre-clinical drug development to clinical trials and assistive technologies for patients. Guests particularly enjoyed getting hands-on extracting DNA from strawberries, using sophisticated microscopes and learning about memory tests as used in the Sheffield memory clinic among many other fascinating demonstrations on offer.
A big Thank-you to all volunteers and helpers on the day and to all our visitors who made the day a fantastic success.
For more images of our Open Day, please head to our album “SITraN Open Day 2015” on our Facebook page. To see some of the feedback and comments of our visitors on Twitter have a look at #SITraN or follow us @neuroshef.
SITraN researchers scooped two prizes at the faculty’s Early Career Prize awards as announced by Professor Chris Newman today on behalf of the Faculty of Medicine, Dentistry & Health (MDH) Early Career Researchers Prize scheme judging panel.
He said: “We had a great number of fantastic nominations from across the Faculty departments for the first year of this new scheme. The judging panel found it extremely difficult to select the winners from those nominated, however I'm thrilled to announce that the following 5 ECRs have been awarded a prize and will receive a grant of £500 for future career development.”
Winners of the Faculty of MDH ECR Prize Scheme 2015 (pictured from left to right, except Dr Paul Morris)
Prof Newman added: “The judging panel would like to thank all nominated researchers for the excellent contributions they have made to the Faculty.”