Promising results published today in the Lancet on Exon Skipping to treat Duchenne Muscular Dystrophy tinged with disappointment as extension study is not undertaken Monday 25 July 2011 PDF Print Delays to development of breakthrough treatments brings bitter disappointment to Duchenne families Nick Catlin, CEO of charity Action Duchenne, the only national charity in the UK focused on finding a cure and raising awareness of Duchenne Muscular Dystrophy commented on results of the AVI Biopharma clinical trials of the morpholino drug AVI-4658 published today in the Lancet: “These results show that we have a potential personalised medicine to treat Duchenne Muscular Dystrophy. Despite the short period of exposure to the Morpholino drug from AVI Biopharma all boys showed some levels of dystrophin expression - one young person at potentially therapeutic levels. It is a considerable disappointment however, that the promised extension study of this trial was not undertaken as this would have given more crucial data over a much longer period of exposure. “Action Duchenne is working closely with AVI Biopharma and the MDEX tream to support ways to move this drug to the market. We will be asking the European Medicines Agency to understand the need to view these Antisense Oligonucleotids drugs as platform medicines so that other gene specific drugs of this type can be designed and trialled without the long time frames and costs. “On behalf of all those living with Duchenne we would like to congratulate Professor Francesco Muntoni and his team for leading this excellent programme of scientific research and discovery.” In a clinical trial of 19 patients, study participants at Great Ormond Street Hospital for Children NHS Trust (GOSH) and the Royal Victoria Infirmary, Newcastle, aged 5 – 15 were given weekly intravenous doses of the study therapy, AVI-4658 which had already been tested for safety and efficacy by the MDEX Consortium and AVI Biopharma in an earlier phase (Kinali et al, Lancet Neural 2009) of the study Parents Claire and Ian Bosanquet whose sons Jack (10) and Tom (7) both have Duchenne with a deletion from 45-50, found both the administrative and clinical research staff at Great Ormond Street Hospital very helpful and efficient. “We got regular updates on the results and the next stages in the research,” said Claire. “Also, as we were attending GOSH regularly, the staff developed an excellent rapport and understanding of the boys and their needs, which really helped with their willingness to cooperate with the trial.” Taking the overall experience into consideration, Claire and Ian both feel very positive about their involvement with the trial; “We feel that there is light at the end of the tunnel, but the research does have a way to go.” James and Dorothy Hetherington who are parents of Ben who is now 11 and who took part in this trial commented, “Our son Ben was 10 when he took part in the trial. Ben was very keen to take part and the care that he received while attending the Royal Victoria Infirmary in Newcastle was excellent. Amazingly he produced the highest % increase in dystrophin fibres, however it has now been 18 months since his treatment ended and seeing him deteriorate so much during this time is heartbreaking. MDEX and AVI must work closely and quickly to get this therapy onto the market before another generation of boys with DMD are no longer with us.” ends- Notes to Editors About Action Duchenne Established in 2001 Action Duchenne aims to support and promote innovative research into a cure and effective medicines for Duchenne/Becker Muscular Dystrophy. The charity, which is led by Duchenne families, aims to promote awareness of the condition, to improve care services, and provide access to a range of educational and support/development programmes for people living with Duchenne at every stage of the condition. This is achieved by working in partnership with government agencies, NHS and care organisations, other charities, academic, scientific and research groups, and biotech companies worldwide. Action Duchenne is actively campaigning for best practice in care for Duchenne based on the publication of an international consensus document in Lancet Neurology in Jan/Feb 2010. The published article was the product of an extensive review by 84 international experts in Duchenne Muscular Dystrophy diagnosis and care, and was supported by the Centers for Disease Control (CDC) in the US. It provides an expert guide to recommendations on the multidisciplinary care that should be available to all individuals with Duchenne. Several Duchenne patient organisations including Action Duchenne and the TREAT-NMD network have helped to produce a ‘Guide for Families’, based on the original academic article, which is written in language accessible to all without a medical background. Since 2003 Action Duchenne has provided £4m for research projects and partnerships. The charity has worked with the MDEX consortium, Department of Health, and the Medical Research Council to deliver new clinical trials for Duchenne drugs. In addition, it has been instrumental in developing projects with biotech companies both in the UK and US including key projects with AVI Biopharma and Summit. In 2005 Action Duchenne launched the DMD Patient Registry, a national database that holds gene information of people living with Duchenne that is used to speed up the recruitment of patients for clinical trials. For more information please visit: www.dmdregistry.org In 2008 Action Duchenne launched the Include Duchenne project in partnership with Decipha. This programme addresses the learning and behaviour needs of those living with Duchenne. For more information please visit: www.decipha.org Action Duchenne promotes social inclusion within the community for young men living with Duchenne through its Genius programme. For more information please visit: www.actionduchenne.org/geniusproject In 2010 the National Advocacy Council was launched to spearhead the lobby for government funding for research into Duchenne and to improve access to Standards of Care. www.actionduchenne.org/NAC For more information please visit: www.actionduchenne.org Editors Contacts Nick Catlin CEO, Action Duchenne Tel: 0208 556 9955 Email: firstname.lastname@example.org Andreina West PR Artistry Limited Tel: 01491 639500 email: Andreina@pra-ltd.co.uk This press release was distributed by ResponseSource Press Release Wire on behalf of PR Artistry in the following categories: Children & Teenagers, Men's Interest, Health, Women's Interest & Beauty, Medical & Pharmaceutical, for more information visit https://pressreleasewire.responsesource.com/about.