Little girl born deaf in UK can now hear after successful gene therapy

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The little girl can now respond to quiet sounds, such as whispers, which has not been the case since birth.

SCIENCE is a promising first step. A little British girl who was deaf from birth learned what it means to hear after the success of a gene therapy she was able to follow as part of a global study welcomed by the British Public Health Service (NHS) this Thursday, May 9. .

Opal Sandy became the first British patient and the youngest child to receive this treatment. The results were also described as “entertaining” from its director. They even better than I expected”“said Professor Manohar Bans from Cambridge University Hospital and principal investigator of the CHORD trial, which began in May 2023.

Four weeks after surgery, an injection into the cochlea (part of the inner ear) under general anesthesia, Opal began to respond to sounds. And after 24 weeks, his hearing was almost normal for quiet sounds, such as whispers.

This little girl was born with a genetic disorder called auditory neuropathy, which is caused by a disruption in the transmission of nerve signals from the inner ear to the brain. The 18-month-old girl now responds to her parents’ voices and can say words such as ” dad “ Or ” Goodbye “.

A major breakthrough for people who are deaf due to an abnormality in the gene that produces otopherin, a protein needed by the hair cells of the inner ear to communicate with the auditory nerve. This anomaly affects about 20,000 people in the UK, Germany, France, Spain and Italy. “We hope this is the beginning of a new era of gene therapy for the inner ear and many types of hearing loss.”added the British researcher.

Promising cases in the US and China

“When Opal could hear us clapping without any help, it was incredible.”his mother Jo Sandy was able to testify. “We were so happy when the clinical team confirmed at 24 weeks that his hearing was picking up softer sounds and speech.”she added.

Positively, this first global study could be used to inform other similar studies that are underway or about to begin in the United States, Europe and China, some of which have already reported positive results.

Earlier this year, Children’s Hospital of Philadelphia announced that an 11-year-old boy “born profoundly deaf” heard “for the first time in my life” after gene therapy. Four months after surgery, the child has only mild to moderate hearing loss.

Additionally, a study published earlier this year in the medical journal Lancet found that similar treatment given to six deaf children in China allowed five of them to regain hearing.

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