British girl has hearing restored as first patient on new gene therapy trial

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Opal, aged 18 months, was treated at Addenbrooke’s Hospital in Cambridge and now has almost normal hearing in her right ear.

A British girl has had her hearing restored after becoming the first person in the world to take part in a groundbreaking new gene therapy trial.

Professor Manohar Bance, an ear surgeon at the trust and chief investigator for the trial, told the PA news agency the results were “better than I hoped or expected” and may cure patients with this type of deafness.He said: “We have results from which are very spectacular – so close to normal hearing restoration. So we do hope it could be a potential cure.”

Her parents Jo and James, both 33, noticed improvements to her hearing in four weeks when Opal turned her head to loud clapping.“I thought it was a fluke or like a change in light or something that had caught her eye, but I repeated it a few times. “We were told she had near normal hearing last time – I think they got responses at sort of 25 to 30 decibels.

A second child has also received the gene therapy treatment at Cambridge University Hospitals, with positive results seen recently, six weeks after surgery. Prof Bance said: “My entire life, gene therapy has been ‘five years away’, and I’ve been in practice about 30 years.“So, for me, it was almost unreal that this moment had arrived.

China has also been working on targeting the same gene, with positive results, though Prof Bance said theirs uses a different technology and slightly different mode of delivery. He said it could take a while for more children to benefit from gene therapy. The treatment was currently not available on the NHS.

“And then we just repair and close up, so it’s actually a very similar approach to a cochlear implant, except we don’t put the implant in.”

 

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