News - A phase l/lla clinical trial in DMD using systemically delivered morpholino antisense oligomer to skip exon 53 - funding from the EU for a collaborative study on the above project under the FP7 health Innovation has been awarded to a consortium to include nine academic institutions and industries in Europe and the USA. Professor Francesco Muntoni at UCL will be the coordinating person for the award. Additional information will be available in due course.

The MDEX Consortium is pleased to announce that Profesor Len Seymour has agreed to succeed Professor Dame Kay Davies as the chair of the Scientific Advisory  Board (SAB). Professor Seymour is a Professor of Gene Therapies and is the Director of the Clinical Pharmacology Section in the Department of Oncology, University of Oxford. The MDEX Consortium is grateful to Professor Dame Kay Davies for chairing the  SAB and the advice and support since 2005.

The MDEX Consortium was formed to develop and test treatments for Duchenne muscular dystrophy. In the last few years the MDEX Consortium has focussed on a strategy of using patches of genetic material (Antisense Oligonucleotides) to restore production of the essential muscle protein dystrophin.The activities of the MDEX Consortium are divided into several sections, each led by a different research group (see profiles) but with all members of the Consortium sharing results and meeting regularly. The project also benefits from regular input from a Scientific Advisory Board of experts (see Scientific Advisory Board).

(PIs and Postdocs at the MDEX Consortium meeting 03.11.11 at UCL/ICH)

Originally the work of the MDEX Consortium was funded by the Department of Health (UK) in 2005. This project was focused on the search of the most effective molecular patch to promote the restoration of dystrophin production in Duchenne boys carrying specific genetic changes (deletions). This culminated in a phase lb/lla clinical trial in a group of boys who received the molecular patch AVI-4658 intramuscularly (Kinali et al, Lancet Neurology 2009). A summary of the outcome can be seen under latest news on this website. This was followed by funding from the MRC UK and the sponsor AVI Biopharma for a dose escalation systemic trial in 19 patients at the Great Ormond Street Hospital (GOSH), London and the Newcastle Royal Infirmary. The data was published in the high impact journal Lancet in 2011.

Link to Lancet paper:http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(11)60756-3/fulltext.View the full press release in our News section.

Additional insight into this study can be viewed in the MRC highligts  http://www.mrc.ac.uk/About/AnnualReview09-10/SevenAges/Childhood/Hopeformuscular/index.htm

The UK North Star Clinical network, which links 20 UK centres involved  in the diagnosis and management of DMD (http://www.muscular-dystrophy.org/how_we_help_you/for_professionals/clinical_databases) were closely associated in the above studies and made referral of patients. Q&A relating to exon skipping can be viewed in this website and the Clinical Trials website http://clinicaltrials.gov/ct2/show/NCT00844597.

The United States Adapted Names Council approved the nonproprietary name ETEPLIRSEN for AVI-4658. Information on clinical trials in the USA relating to AVI-4658 is now available from David Walsey, Head of Invester Relations and Corporate Communications, AVI BioPharma. ( This e-mail address is being protected from spam bots, you need JavaScript enabled to view it )

Funding Awards: The MDEX Consortium has been succesful in obtaing two prestigious awards (1) from the Department of Health and the Wellcome Trust for a period of 36 months totalling up to nearly £2.5 Million for the study 'Advanced antisense oligonucleotide technology for exon skipping in Duchenne muscular dystropy' and (2) from Association Francaise contre des Myopathies (AFM) for a period of 48 months totalling upto €3.9 Million for the study 'Advances in oligonucleotide-mediated exon skipping for DMD and related disorders'. Both awards were to Professors Matthew Wood and Francesco Muntoni. The awards will facilitate research in the laboratories of Professor Matthew Wood (Oxford), Professor Francesco Muntoni/Dr Jenny Morgan/Dr Steve Hart (UCL/ICH), Professors Kate Bushby/Volker Straub (Newcastle), Professor George Dickson (Royal Holloway), Professor Dominic Wells (Royal Veterinary), Dr Mike Gait (Cambridge), Professor Bernard Lebleu (Montpellier) and Professor Thomas Voit (Paris). The awards include funding for clinical trials in year 2 onward in London and Newcastle. More details on the awards will be available in our News section in due course.

GSK/Prosensa clinical trial in DMD boys with study drug GSK2402968 (PRO051): A multicentre trial with this study drug recruited DMD boys in UK at the Great Ormond Street Hospital(GOSH), London and at the Royal Victoria Infirmary (RVI), Newcastle. It is a Phase lla, double blind, exploratory, parallel clinical trial to assess the optimal dose of GSK2402968 for safety, tolerability and efficacy, in ambulant patients with DMD. A total of 10 DMD boys were recruited in the UK, 5 at the RVI and 5 at GOSH. This study is now closed for recruitment. Four boys from each of the above sites have now enrolled into the extension study after completing their initial treatment.

TROPHOS-Olesoxime study in SMA - This is a phase ll, multicentre, randomised placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old spinal muscular atrophy (SMA) patients. In the UK the study will be at GOSH London, Royal Victoria Infirmary Newcastle and Birmingham Heartlands Hospital. Other than the centres in the UK , the study recruited patients in Belgium, France, Germany, Italy, the Netherlands and Poland. The study is now closed for recruitment at all sites.

The DMD Heart Protection Study has commenced in Newcastle and in GOSH, London. Nine patients from each site have been recruited and recruitment is ongoing. Please contact Dr Mariacristina Scoto, Clinical Research Fellow on 020 7905 2639 for further details. Additional details with inclusion criteria is available under the news section on this website.

Ataluren (PTC124) PTC Therapeutics: The preliminary findings from the Ataluren Study 007 did not show significant muscle improvement in the patients who participated in the study. The study was therefore discontinued. An update on this study was presented at the International Congress on Neuromuscular Diseases, Naples, Italy, 17-22 July 2010 and details are available on www.ptcbio.com.

PTC has taken full reponsibility for the study drug Ataluren worldwide and are in the process of extending the follow-on study to Europe. They have contracted REGISTRAT-MAPI (EU), Lyon, France as the CRO in charge of providing  all the necessary management activities to initiate the follow-on study, subject to regulatory approval.