“Spectacular” results: a baby born deaf hears thanks to gene therapy

Une petite fille britannique née sourde peut désormais entendre grâce à une thérapie génique. Tutye/Getty Images

Une petite fille britannique née sourde peut désormais entendre grâce à une thérapie génique dans le cadre d’un essai aux résultats salués comme "spectaculaires" par son directeur.

Opal Sandy is the first patient treated in a global gene therapy trial, the British public health service said.

She is the first British patient and the youngest child to receive this type of treatment.

The little girl was born with a genetic disease, auditory neuropathy, caused by a disruption of nerve signals from the inner ear to the brain.

She responds to her parents' voice

Around 20,000 people in the UK, Germany, France, Spain and Italy are deaf due to an abnormality in a gene that produces otoferin, a protein needed by cells ciliated cells of the inner ear to communicate with the auditory nerve.

In the four weeks following the operation, an injection into the cochlea (part of the inner ear) under general anesthesia, Opal reacted 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 pronounce words like "daddy" or "goodbye".

A "new era"

"When Opal could hear us clapping our hands without help, it’was incredible,” testified her mother Jo Sandy. "We were so happy when the clinical team confirmed at 24 weeks that her 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 loss “hearing,” he added.

Other similar studies are underway or about to begin at the beginning of the year in the United States, Europe and China, some of which have been successful.< /p>

At 11 years old, he hears "for the first time in his life"

Earlier this year, Children's Hospital of Philadelphia announced that an 11-year-old boy "born profoundly deaf" heard "for the first time in his life" after gene therapy and four months after the operation, the child only has mild to moderate hearing loss.

A study published at the beginning of the year in the medical journal The Lancet revealed that a similar treatment administered in China to six deaf children had enabled five of them to regain hearing.