baby born deaf hears thanks to gene therapy

The head of this trial is delighted with the “spectacular” results and hopes that it opens “a new era for gene therapies for the ear”.

At 18 months old, little Opal Sandy is the youngest child to receive this type of treatment for deafness. This British girl suffering from a genetic disease can now hear thanks to gene therapy, the public health service in the United Kingdom (NHS) announced on Thursday, May 9. “These results are spectacular and better than I expected” said Professor Manohar Bance, of Cambridge University Hospital, chief investigator of a global trial called CHORD, which began in May 2023.

This infant suffers from auditory neuropathy caused by a disruption of nerve signals from the inner ear to the brain. The first to test this protocol, he received an injection into the cochlea (part of the inner ear) under general anesthesia. Four weeks after the operation, Opal Sandy was responding to sounds, the NHS explains. 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 “dad” Or “Goodbye”.

“This is, we hope, the start of a new era for gene therapies for the inner ear and many types of hearing loss,” comments Manohar Bance. Around 20,000 people in the UK, Germany, France, Spain and Italy have deafness due to a defect in a gene that produces otoferin, a protein needed by hair cells in the ear internal to communicate with the auditory nerve.

Other innovative treatments around the world

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” heard “for the first time in his life” after gene therapy. 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 allowed five of them to regain their hearing.