Leading RNA-based biopharmaceutical company announces successful clinical trials for treatment of muscle wasting disease by ‘exon skipping’
Action Duchenne states that the latest results of a study released by AVI BioPharma Inc, a developer of RNA-based drugs, demonstrate that Duchenne Muscular Dystrophy could be treated successfully by exon skipping. The clinical trial of the drug AVI-4658, developed by AVI BioPharma, was funded by the Department of Health (UK) and conducted by the members of the MDEX* consortium, led by Professor Muntoni at UCL Institute of Child Health London. Biopsy data showed that injection of the drug into the foot muscles of a series of Duchenne patients successfully induced new dystrophin production. The study data will also be published in a specialist medical journal.
Recruitment of Duchenne patients for a further AVI-sponsored trial to see if this drug can be delivered body wide is now underway. The MDEX team will be using Action Duchenne’s DMD Registry www.dmdregistry.org to help in recruiting Duchenne boys with specific gene variations for this new clinical trial. The trial, which was opened in December 2008, will administer AVI-4658 systemically to see if dystrophin can be produced in all muscles. It is hoped that this might slow the very severe progression of the disease.
Duchenne Muscular Dystrophy affects 1 in 3,500 male births in the UK, and is the most common and severe type of muscular dystrophy - sufferers are diagnosed usually by the age of 5. Patients with DMD and Becker Muscular Dystrophy (BMD) are boys and young men who lack dystrophin, a protein that is critical to the structural stability of muscle fibres. Patients develop progressive muscle weakness. Duchenne affects all muscles including the heart and respiratory system leaving young people paralysed by late teens and many patients do not live past their twenties.
Recent research by the MDEX Consortium has demonstrated that short molecules – called antisense oligonucleotides (AOs) – can restore the production of dystrophin protein to affected tissues. This is done by a process called ‘exon skipping’, in which the parts of the genetic code affected by a mutation are “patched” by AO’s so that that the code can be correctly read.
This latest drug AVI–4658 is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. Restoration of dystrophin production achieved by skipping this exon may potentially improve or significantly slow the disease process, thus prolonging and improving the quality of life for the affected patient.
Nick Catlin, CEO of Action Duchenne said, “These early results that show exon skipping therapy can be a potential treatment for Duchenne Muscular Dystrophy is promising news that will be welcomed by all sufferers and their families. The MDEX Consortium has made some tremendous progress in researching treatments for this disease and this latest great work underlines the value of continuing to actively raise funds to support this important cause.”
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Last updated on: 27/08/2010 11:40:18