Deaf toddler has hearing restored after world first gene therapy trial that could be 'potential cure'

9 May 2024, 09:56 | Updated: 9 May 2024, 10:00

Deaf toddler has hearing restored after world first gene therapy trial that could be 'potential cure'
Deaf toddler has hearing restored after world first gene therapy trial that could be 'potential cure'. Picture: Alamy

By Christian Oliver

A deaf toddler can now hear after becoming the first person in the world to take part in a new gene therapy trial.

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Opal Sandy, an 18-month-old from Oxfordshire, was born completely deaf due to a condition of auditory neuropathy - an issue that prevents nerve impulses travelling from the inner ear to the brain.

But the toddler's hearing is now almost normal - and could improve even further - following the gene therapy trial in the UK and worldwide.

Professor Manohar Bance, an ear surgeon and chief investigator for the trial at Cambridge University Hospitals NHS Foundation Trust trust told the PA news agency that the results were "better than I hoped or expected".

He said the successful trial means patients with this type of deafness may now all be cured.

He said: "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."

Opal Sandy can now hear unaided for the first time after receiving ground-breaking gene therapy
Opal Sandy can now hear unaided for the first time after receiving ground-breaking gene therapy. Picture: Alamy

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The "new era" gene therapy - from biotech firm Regeneron - involved Opal receiving an infusion containing a working gene in her right ear during surgery last September.

Her parents Jo and James, both 33, noticed improvements to her hearing in four weeks when Opal turned her head to loud clapping.

"When she first turned, I couldn't believe it," Mrs Sandy told PA. "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.

"I picked my phone up and texted James, and said 'I think it's working'. I was absolutely gobsmacked. I thought it was a fluke."

But results improved even further around 24 weeks after surgery when tests in Cambridge showed Opal could also hear soft sounds such as a whisper.

"The audiologist played back some of the sounds that she was responding to and they were ridiculously quiet sort of sounds that in the real world wouldn't catch your attention during a conversation," Mrs Sandy said.

"Certainly since February, we've noticed her sister (Nora) waking her up in the morning because she's running around on the landing, or someone rings on the door so her nap's cut short.

"She's definitely responding more to sort of what we would call functional sounds rather than just sounds that we use to test her.

"We were told she had near normal hearing last time - I think they got responses at sort of 25 to 30 decibels.

"I think normal hearing is classed at 20 decibels, so she's not far off. Before, she had no hearing whatsoever."

Opal Sandy (centre), who was born completely deaf because of a rare genetic condition, with her family
Opal Sandy (centre), who was born completely deaf because of a rare genetic condition, with her family. Picture: Alamy

Prof Bance said Opal's hearing is now "close to normal" and said he hopes "she'll get back to normal by the next testing."

He said the treatment is "a one-and-done therapy, so hopefully you have your treatment and then you go back to your life".

A second child has also received the gene therapy treatment at Cambridge University Hospitals, with positive results seen recently, six weeks after surgery.

Up to 18 young people have been recruited for the trial from the UK, Spain, and the US. They will be followed up for five years.

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.

"It was just the fact that we've been hearing about this for so long, and there's been so much work, decades of work ... to finally see something that actually worked in humans ... It was quite spectacular and a bit awe-inspiring really.

"It felt very special."

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