A new gene therapy has been trialled in the UK, restoring the hearing of an 18 month old girl.
Opal Sandy was born deaf due to a condition called auditory neuropathy. This is caused by the disruption of nerve impulses travelling from the inner ear to the brain.
Thanks to a ‘one hit’ gene therapy, the little girl is now completely cured, her hearing has been restored.
Her parents, Jo and James were “gobsmacked” when she responded to sound tests.
“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.
Experts are also delighted. Professor Manohar Bance, an ear surgeon at the trust and chief investigator for the trial, said the results were “better than I hoped or expected”, and may cure patients with this type of deafness.
So how does it work? It’s pretty simple according to Professor Bance.
“The inner ear was opened and the treatment infused using a catheter over 16 minutes.
“We have to make a release hole in another part of the ear to let the treatment out because it has to go all the way through the ear,” he said.
“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.”
So what’s next?
The trial is in three stages. This is the first where children, like Opal are exposed to a low dose in just one ear.
Another three children will then get a high dose on one side. Then, if safe, a new set of children will get a dose in both ears at the same time.
Up to 18 people from the UK, US and Spain are being recruited and this will be followed for five years.
Opal is happy and hearing. Her parents report that she loves the sound of clicking cutlery and her wooden drum.
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HeadlineGirl, deaf since birth, has hearing restored in breakthrough trial
Short HeadlineGene therapy restores deaf girl's hearing
StandfirstA new gene therapy has been trialled in the UK, restoring the hearing of an 18 month old girl.
A new gene therapy has been trialled in the UK, restoring the hearing of an 18 month old girl.
Opal Sandy was born deaf due to a condition called auditory neuropathy. This is caused by the disruption of nerve impulses travelling from the inner ear to the brain.
Thanks to a ‘one hit’ gene therapy, the little girl is now completely cured, her hearing has been restored.
Her parents, Jo and James were “gobsmacked” when she responded to sound tests.
“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.
Experts are also delighted. Professor Manohar Bance, an ear surgeon at the trust and chief investigator for the trial, said the results were “better than I hoped or expected”, and may cure patients with this type of deafness.
So how does it work? It’s pretty simple according to Professor Bance.
“The inner ear was opened and the treatment infused using a catheter over 16 minutes.
“We have to make a release hole in another part of the ear to let the treatment out because it has to go all the way through the ear,” he said.
“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.”
So what’s next?
The trial is in three stages. This is the first where children, like Opal are exposed to a low dose in just one ear.
Another three children will then get a high dose on one side. Then, if safe, a new set of children will get a dose in both ears at the same time.
Up to 18 people from the UK, US and Spain are being recruited and this will be followed for five years.
Opal is happy and hearing. Her parents report that she loves the sound of clicking cutlery and her wooden drum.
