This year’s Annual Conference research talk was given by Dr Chris McDermott and focused on his research at the Sheffield Institute for Translational Neuroscience (SiTraN). His talk, adequately named ‘Improving the care and evidence base for symptomatic care’, was a very personal and informative talk to all that attended.
Dr McDermott gave a brief introduction about how he became a neurologist and got involved with MND research. He then talked about non-invasive ventilation (NIV) and how, through healthcare research, they have already gained evidence that NIV increases survival and improves the quality of life for people with MND. After this evidence was obtained, the technique gained National Institute of Health and Clinical Excellence (NICE) approval and NIV is now a standard treatment for people with MND who experience respiratory weakness.
Dr McDermott also mentioned how a ‘cough assist machine’ can be used to help patients cough and get rid of secretions and dirt. Information from the literature suggested a reduction of hospital admissions, and infections, in the year following the use of a cough assist machine for other diseases. The results from Dr McDermott’s MND Association funded study, looking at a cough assist machine for people with MND are yet to be analysed.
The rest of Dr McDermott’s talk focused on his current diaphragm pacing trial (known as DiPALS) being part-funded by the MND Association.
Dr McDermott explained that diaphragm pacing is designed so that people with MND do not have to be on long-term ventilation units, as the remote unit can be carried around when in use. Diaphragm pacing is similar to that of a ‘pace maker’, but instead of the heart it stimulates the diaphragm using an electrical current.
A video was shown of how the instrument’s electrodes are placed onto the diaphragm muscle during a 30 minute key-hole surgery procedure. The pacing unit is then controlled by an external remote at present, but if trials are successful an internal unit could be developed.
Dr McDermott stressed that the mechanisms in which diaphragm pacing acts is not known at present but suggested that it may cause stronger contractions or restore co-ordinated breathing.
An American study of 106 people with MND who had advanced respiratory problems previously found that 81% of trial participants were still alive after one year. Dr McDermott went on to explain that this study had no control group to test whether the diaphragm pacing unit improves life over and above that of current standards of care. However, the American study did find diaphragm pacing to be safe.
The DiPALS study in the UK has been recruiting patients with MND since November 2011 and compares NIV alone (control group) and NIV plus diaphragm pacing. Dr McDermott’s study aims to see if there is a difference in survival between the two groups, whether there is an improvement to quality of life, whether it is less intrusive, and whether the treatment is cost-effective. If diaphragm pacing does show a difference, then this could lead to NICE approval and become standard NHS-provided care in the UK. Dr McDermott went on to say that even if the study is found not to be a viable treatment the trial has already importantly shown that people with MND can have a general anaesthetic.
As the study is ongoing the final results are not available. Results are due in autumn 2014.
Overall, it was great to have an update into Dr McDermott’s research which stimulated an abundance of questions from all those that attended, leaving delegates with a real insight into some of the healthcare research funded by the Association.
If you’re interested in attending a future MND Association conference to hear the latest about MND research, look out for our announcement of our 2013 Spring Conferences. Dates and locations will be announced on our website www.mndassociation.org.
Diaphragm pacing trial information sheet
AGM and Annual Conference summary on our website: http://www.mndassociation.org/news-and-events/events/conferences/AGM+and+Annual+Conference+2012
If diaphragm pacing has FDA approval in th US and has been in use there for some time now then why do we need to waste precious time money and lives doing this all over again? John
Hi John, Thanks for commenting.
American FDA approval means that the diaphragm pacing units don’t pose “an unreasonable or significant risk of illness or injury, and that the probable benefit to health outweighs the risk of injury or illness from its use.” It hasn’t yet been tested for its effectiveness over and above that of standard treatment for respiratory weakness such as NIV. This is why the UK trial is needed. Our trial is the first randomized and controlled trial in the world – there’s also a new trial being set up in America to test the effectiveness of diaphragm pacing: http://www.alsa.org/news/the-als-association-partners.html
It’s important that we gain evidence to find out whether diaphragm pacing is more effective than standard treatment options in order for it to be approved by NICE and to become a standard care option. Approval by NICE allows access to treatments across the country and moves us away from a ‘postcode lottery’ system of provision. Without this evidence, and without running these trials we simply won’t be able to do this.
In order to ensure that our funds are used wisely, we are part-funding this trial with the National Institute for Health Research (NIRH). Our leading reputation means that for every £1 we commit to collaborative MND research, we attract another £2 to £3 from other major funding bodies. Through this collaboration with the NIHR, we are paying for the diaphragm pacing units which cost £13k each, and the NIHR are funding the rest of the trial to cover clinician’s time, surgury costs, data analysis etc.
Thanks again for commenting – I hope that this information has clarified why we need the UK diaphragm pacing clinical trial.
Research Development Team
I thought the FDA required phase 1 ,2 and 3 trials addressing safety , tolerance and efficacy before granting approval for treatment. I am obviously mistaken.
Thanks very much for your response.
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