May 9, 2024, 09:56 | Updated: May 9, 2024, 10:00
A hearing-impaired toddler has regained his hearing after the world’s first gene therapy trial offering a ‘potential cure’.
Photo: Alamy
A deaf toddler has become the first person in the world to be able to hear after taking part in a new gene therapy trial.
Opal Sandy, an 18-month-old from Oxfordshire, was born completely deaf with auditory neuropathy, which prevents nerve impulses from passing from the inner ear to the brain.
But as a result of gene therapy trials in the UK and around the world, hearing in young children is now almost normal, and could improve further.
Professor Manohar Vance, ear surgeon at Cambridge University Hospitals NHS Foundation Trust and principal investigator on the trial, told the PA news agency that the results were “better than I expected”.
He said the success of the trial meant all patients with this type of hearing loss could be cured.
He said: “The results[with Opal]were very impressive and very close to restoring normal hearing, so we’re hopeful that this could be a potential treatment.” .
Opal Sandy was able to hear on her own for the first time after undergoing ground-breaking gene therapy.
Photo: Alamy
read more: The father of the “hero” who fought off the Hainaut attacker says a “miracle” saved his family who were sure they would die.
read more: Departure from Conservative Party grows as former prime minister Nadhim Zahawi steps down at next election in latest blow to Rishi Sunak
The “new age” gene therapy by biotech company Regeneron included Opal receiving an IV containing the activated gene in her right ear during surgery last September.
Opal’s parents, Joe and James, both 33, noticed that her hearing had improved over the course of four weeks when Opal faced the loud applause.
“When she turned around for the first time, I couldn’t believe it,” Sandy said in Pennsylvania. “I thought it was a fluke or a change in the light or something that caught her eye, but I repeated it a few times.
“I picked up my phone and texted James and said, ‘I think it’s going well.’ I was completely taken aback. I thought it was a fluke.”
But results improved further around 24 weeks after surgery, when tests in Cambridge showed Opal could hear even soft sounds, such as whispers.
“The audiologist played some of the sounds she was responding to, but they were incredibly quiet sounds that wouldn’t attract attention in real life during a conversation,” Sandy said.
“Indeed, since February, we have noticed her younger sister (Nora) waking up in the mornings because she is running around on the landing or someone rings the door and her naps are cut short.
“She’s definitely responding more to so-called functional sounds rather than just the sounds we use to test her.
“Last time, we were told that her hearing was almost normal, but I think it was probably around 25 to 30 decibels.
“I think normal hearing classifies as 20 decibels. So she’s not that far off. Before, she had no hearing at all.”
Opal Sandy (center), who was born completely deaf due to a rare genetic disease, and her family.
Photo: Alamy
Professor Vance said Ms Opal’s hearing was now “approaching normal” and “we hope it will return to normal by the time of her next test”.
He said the treatment is “a one-time treatment, so we hope that he can get back to his normal life after receiving the treatment.”
Their second child also underwent gene therapy at Cambridge University Hospital and recently saw positive results six weeks after surgery.
The trial recruited up to 18 young people from the UK, Spain and the US. They will be followed for five years.
Professor Vance said, “In my lifetime, gene therapy is “5 years away,” and I have been practicing it for about 30 years.
“So for me, it was almost surreal that this moment came.
“It’s true that we’ve been hearing about this for a long time, and there’s been so much research and decades of research…to figure out what actually works in humans. I finally got to see it…it was quite spectacular and a bit of a surprise, really awe-inspiring.
“It felt very special.”