Opal Sandy is the first patient treated as part of a global gene therapy trial, the NHS, the British public health service, said in a statement released Thursday.
She is the first British patient and the youngest child to receive this type of treatment.
The little girl was born with a genetic condition, auditory neuropathy, caused by a disruption of nerve signals from the inner ear to the brain.
20,000 people affected by deafness due to a gene abnormality
Around 20,000 people in the UK, Germany, France, Spain and Italy are deaf due to a defect in a gene that produces otoferin, a protein needed by hair cells in the inner ear to communicate with the ear. auditory nerve.
Within four weeks of the operation, an injection into the cochlea (part of the inner ear) under general anesthesia, Opal was responding to sounds. After 24 weeks, his hearing was almost normal for soft sounds, such as whispers.
Now 18 months old, the little girl responds to her parents’ voices and can say words like “daddy” or “goodbye.”
“It was incredible”
“When Opal could hear us clap our hands without help, it was incredible”testified his mother Jo Sandy. “We were so happy when the clinical team confirmed at 24 weeks that his hearing was also picking up softer sounds and speech,” she added.
“These results are spectacular and better than I expected”said Professor Manohar Bance, of Cambridge University Hospital and chief investigator of the CHORD trial, which began in May 2023.
“This is, we hope, the start of a new era for gene therapies for the inner ear and many types of hearing loss.”he added.
Other similar studies are underway or about to begin earlier this year in the United States, 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. The child now only has mild to moderate hearing loss.
A study published earlier this year in the medical journal The Lancet revealed that a similar treatment administered in China to six deaf children allowed five of them to regain their hearing.
