A little British girl born deaf can now hear thanks to gene therapy as part of a trial with results hailed as “spectacular” by its director. 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.
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 auditory nerve.
“Spectacular results”
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.”
“When Opal could hear us clapping our hands without help, it was incredible,” said her 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.
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“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. “It’s “We hope the beginning of a new era for gene therapies for the inner ear and many types of hearing loss,” he added.
Other cases
Other similar studies are underway or were 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, l The child now only has mild to moderate hearing loss.
