Clinical trial for sufferers of muscular dystrophy
A team at Newcastle University are beginning a trial this week that could give hope to thousands of sufferers of muscular dystrophy.
A new treatment called molecular patch therapy has been developed which has the potential to give boys born with Duchenne Muscular Dystrophy (DMD) the chance to preserve their muscle function and live into old age.
In a world first, the antisense oligonucleotide (AO) patches work by masking the faulty part of the gene (exon 51) and allowing shortened but functional proteins to be formed.
DMD affects one in 3,500 boys and is caused by reduced production of dystrophin protein - vital for muscle function. The progression of the condition is so severe that untreated boys lose the ability to walk by their early teens are only expected to live into their twenties.
From the Newcastle University website:
“This is a major breakthrough for the treatment of DMD,” said Professor Francesco Muntoni, head of the neuromuscular unit at Imperial College Healthcare NHS Trust, “as conventional gene therapy approach for this disorder has proven to be problematic. Animal work has suggested that the molecular patch has worked well and showed a very significant restoration in dystrophin function”.
Professor Kate Bushby and Professor Volker Straub from the Institute of Human Genetics at Newcastle University will be recruiting nine young men from across the country and preparing them for the treatment. The trial participants will need to be aged 12 – 17 with DMD and they will have the molecular patch administered by injection into a small muscle in the foot. Subject to the trial’s success, there is already a plan to proceed with another trial to deliver the molecular patch under the skin, so that all muscles in the body could be treated.
Professor Bushby of Newcastle University said ”This is very important work to be involved with and a reflection of the long -standing interest in muscular disease here in Newcastle. We’re one of only three specialist centres in the country and this is the first step to what might be a gene based therapy for Duchenne Muscular Dystrophy.”
