“When Opal could hear us clapping without any help, it was incredible,” said Jo Sandy, her mother. “We were so happy when the clinical team confirmed at 24 weeks that his hearing was picking up softer sounds and speech,” she added.
A little British girl who was born deaf can now hear thanks to gene therapy, in a study whose results have been described as “impressive”. Opal Sandy is the first patient to be treated in a global gene therapy trial, Britain’s National Health Service said in a statement on Thursday.
She is the first British patient and the youngest child to receive this treatment. The little girl was born with a genetic disorder called auditory neuropathy, caused by a disruption in the transmission of nerve signals from the inner ear to the brain.
The now 18-month-old little girl responds to her parents’ voices and can say words like “daddy” or “goodbye.” For her parents, the moment the baby was able to hear them was “mind-blowing,” they told the Daily Mirror.
About 20,000 people in the UK, Germany, France, Spain and Italy are deaf due to a defect in the gene that produces otopherin, a protein needed by the hair cells of the inner ear to communicate with the auditory nerve.
Within four weeks of the surgery (an injection into the cochlea (part of the inner ear) under general anesthesia), Opal began to respond to sounds. After 24 weeks, his hearing was almost normal for quiet sounds such as whispers. The now 18-month-old little girl responds to her parents’ voices and can say words like “daddy” or “goodbye.”
“A New Era of Inner Ear Gene Therapy”
“These results are exciting and better than I expected,” said Professor Manohar Bans from Cambridge University Hospital and principal investigator of the CHORD trial, which began in May 2023. “We hope this is the beginning of a new era of gene therapy for the inner ear and many types of hearing loss,” he added. Other similar studies are underway or set to begin early this year in the US, Europe and China, some of which have been successful.
Earlier this year, Children’s Hospital of Philadelphia announced that an 11-year-old boy “born profoundly deaf” was hearing “for the first time in his life” after gene therapy and four months after surgery. moderate hearing loss.
A study published earlier this year in the medical journal The Lancet found that similar treatment given to six deaf children in China restored hearing to five of them.
Source: Le Parisien
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