UK Neuromuscular Translational Research Conference 2009
On March 26 and 27 scientists and clinicians gathered in Newcastle for the UK Neuromuscular Translational Research Conference. This is the second time that this annual meeting which is jointly organised by the Muscular Dystrophy Campaign and the MRC Centre for Neuromuscular Diseases has been held. The programme included international experts in the field of muscle disease, with some 200 clinicians, scientists, industry partners, representatives from government organisations and patients’ groups attending.
The conference built on the success of last year’s meeting with the latest results of exciting new potential treatments being presented and discussed. Of great interest was the presentation for the first time of the results of the MDEX exon skipping clinical trial for Duchenne muscular dystrophy by Prof Francesco Muntoni (see previous page). Gerard Platenburg from Prosensa also presented the latest results from their exon skipping trial which is ongoing in the Netherlands. Both of the trials have produced promising initial results proving that injecting a muscle with this new therapy could induce the production of dystrophin- the protein that is missing in boys with Duchenne muscular dystrophy. They are both now conducting further trials to attempt to deliver the therapy to all of the muscles of the body. The two trials are testing slightly different chemical formulations of the exon skipping technology.
Some of the highlights of the meeting were talks presented by Muscular Dystrophy Campaign grantees. In particular, Prof. David Rubinsztein from the University of Cambridge presented some promising treatment approaches for oculopharyngeal muscular dystrophy which have been unlocked by his detailed study of the mechanism causing this condition. Dr Mary Reilly also reported on the progress of their trial of Vitamin C for Charcot-Marie-Tooth disease which is due to finish in the middle of this year. Four talks on exercise therapy for muscle disease highlighted the value of this treatment approach for many disorders including mitochondrial myopathy, fascioscapulohumeral muscular dystrophy (FSHD) and McArdle disease. They also called for more research in this area to determine how much and what type of exercise is the most effective and safe.
The MRC Centre for Neuromuscular Diseases is a joint venture between University College London and the University of Newcastle-upon-Tyne which was set up to address the various barriers that currently hinder a speedy ‘bench-to-bedside’ transfer of technology. As such, several talks addressed issues in this area including the most suitable animal models to test treatment approaches and standardised methods for doing this research.
The progress of the Smartnet Clinical Network for spinal muscular atrophy was also reported. This network is jointly funded by the Muscular Dystrophy Campaign and Jennifer Trust for Spinal Muscular Atrophy and aims to improve standards of care as well as gather patient information for a database that is vital for the setting up clinical trials in the future.
Professor Dame Kay Davies gave the first John Walton Lecture. She presented an excellent overview of the potential therapies for Duchenne muscular dystrophy, including her own laboratory’s work on utrophin. Kay’s work has been funded by the Muscular Dystrophy Campaign for over 25 years and in her talk she acknowledged the importance of this support. The lecture honoured Lord Walton, one of the founders of the charity, who said he was thrilled to have Kay deliver the first of these lectures.
As well as a busy schedule of 24 talks over the two days, the latest results of 68 research projects were also displayed- about one third of which are funded by the Muscular Dystrophy Campaign. These were scrutinised during tea and lunch breaks with prizes being given to the best two posters (sponsored by the Muscular Dystrophy Campaign and the MRC Centre for Neuromuscular Diseases).
If you would like to see a copy of the programme from the conference please get in contact with the research department:
