North East will be involved in future muscular dystrophy trials

REASSURANCES have been given that clinical trials in the North involving children with a deadly muscle wasting disease will be followed up.

REASSURANCES have been given that clinical trials in the North involving children with a deadly muscle wasting disease will be followed up.

Researchers in Newcastle contributed to a study that gave children with Duchenne muscular dystrophy (DMD) hope for the future.

Seven of the 19 children involved in the study responded to an injection from a drug with the potential to reduce the symptoms of the disease. The charity Action Duchenne has welcomed the results of the trial but expressed concern that the trial has not been extended further.

But academics involved in the trials have told The Journal that the next stages of the process will take place.

Action Duchenne chief executive Nick Catlin said: “These results show that we have a potential personalised medicine to treat Duchenne Muscular Dystrophy.

“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.”

Professor Volker Straub, from Newcastle University, said the team that took part in the research alongside the Royal Victoria Infirmary was “world leading”.

He added: “This was a collaborative project between Newcastle University and the University College London and the trial that has made the headlines included 19 patients, seven of those came from Newcastle or our catchment area.

“If you compare it to what has been offered to the patients so far this is very substantial. It is the first time the disease is really tackled. This is the first step and it will give families and patients hope.

“The next step is finding out whether long-term usage of this drug would be beneficial.

“This trial was a so-called phase two study, the next phase will test the drug against the placebo. If it then shows that the drug compared to the placebo is effective and the boys benefit then hopefully we will get marketing approval.

“Newcastle will definitely be involved in any future trial. There are no clear time plans but I want it to be as quickly as possible but in the end this drug needs to be produced.

“We want it to be a larger study than just 19, it will be an international trial and we need to guarantee we have sufficient drugs, that’s the bottleneck.”

Prof Straub also said further analysis would take place to see if the drug could tackle other genetic diseases.

Around 100 new cases of DMD emerge in the UK each year. The hereditary disease affects one in 3,500 boys born.

Their muscle cells are affected by a lack of the protein dystrophin and by the age of 12 sufferers are left unable to walk. The condition can become life threatening by the late teens or early twenties when breathing is compromised. Three of the boys given higher doses of the drug in the trial had their dystrophin restored to levels up to 18% that of normal muscle.


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