Little Opal Sandy from Britain has become the first deaf person from birth to regain her hearing thanks to a pioneering gene therapy that was implemented as part of a global trial, the NHS has announced. Its results were described as “spectacular” by the head of the study. It is a development that, according to doctors, marks a new era in the treatment of deafness.
The little girl was born with a genetic disease, an auditory neuropathy, caused by the disruption of the transmission of nerve signals from the inner ear to the brain.
About 20,000 people in the UK, Germany, France, Spain and Italy suffer from the abnormality of a gene that produces otoferrin, a protein needed in the hair cells of the inner ear to communicate with the auditory nerve.
At four weeks after the operation, which involves an injection into the cochlea (part of the inner ear) under general anaesthetic, Opal was responding to sounds. After 24 weeks, her hearing was almost normal to soft sounds, such as humming.
The adorable moment deaf toddler Opal Sandy hears for the first time following a world first gene therapy trial.
Opal’s parents Jo and James say the moment was ‘mind blowing.’ pic.twitter.com/S1h5kJEVuz
— The Mirror (@DailyMirror) May 9, 2024
He began to form words
Today, 18-month-old Opal responds to her parents’ voice and can say words like “daddy” and “hello.”
“When Opal was able to hear us without the help of clapping, it was incredible. We were so happy when the clinical team confirmed at 24 weeks that her hearing was picking up softer sounds and speech,” said her mother.
“These results are spectacular and better than I had hoped,” said Professor Manohar Bunce of University Hospital Cambridge and lead investigator of the CHORD trial, which started in May 2023. “We hope it is the start of a new era for genetic treatments of the middle ear and many types of hearing loss’.
Other similar studies are underway or started earlier this year in the United States, Europe and China. Some have positive results.
Earlier this year, the Children’s Hospital of Philadelphia in the US announced that an 11-year-old boy “born with profound deafness” had heard “for the first time in his life” after gene therapy and, four months after the operation, the child had no symptoms. no more mild or moderate hearing loss.
A study published earlier this year by The Lancet found that similar treatment in China on six deaf children allowed five of them to regain hearing.
Source: RES-MPE
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