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A British girl has had her hearing restored after becoming the first person worldwide to take part in a pioneering new gene therapy trial.
Opal Sandy was born totally deaf due to auditory neuropathy, which disrupts nerve impulses from the inner ear to the brain.
But the 18-month-old’s hearing is now almost normal – and could improve further – after having the one-time treatment.
Opal was treated at Addenbrooke’s Hospital in Cambridge and the head of the trial, Professor Manohar Bance, said results were “better than I hoped or expected” and he hopes medics might be able to cure others with this type of deafness.
“We have results from (Opal) which are very spectacular – so close to normal hearing restoration. So we do hope it could be a potential cure,” he said.
Auditory neuropathy can be caused by a fault in the OTOF gene, which makes a protein called otoferlin and enables cells in the ear to communicate with the hearing nerve.
Opal, from Oxfordshire, had an infusion of the working gene into her right ear during surgery in September – the treatment developed by biotech firm Regeneron.
Her parents noticed changes in only four weeks and the improvements were especially noticeable 24 weeks later.
Jo and James Sandy, both 33, said they were “gobsmacked” when she responded to sound tests at home without her cochlear implant, the usual way to treat the condition.
Opal had her operation in September. Pic: Cambridge University NHS Trust
“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,” said Mrs Sandy.
They say she now enjoys the sound of slamming her cutlery on the table and playing with toy drums and wooden blocks.
“We were told she had near…
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