“New era in the treatment of deafness”: child begins to hear after gene therapy | Medicine

Auditory neuropathy is a condition that results from a failure in the conduction of nerve impulses that carry sound information from the inner ear to the brain. This disturbance in Opal Sandy’s hearing, aged 18 months, caused the child to be deaf from birth. Thanks to an innovative gene therapy, the girl from Oxfordshire, England, can now hear.

It took six months for Opal, in addition to being able to hear soft sounds, like whispers, to be able to say words like “mommy” and “daddy”. How does it count The Guardian, the girl’s parents “couldn’t believe it”. “It was crazy,” said Jo Sandy, the child’s mother.

The treatment was developed at Addenbrooke’s Hospital, a university hospital unit and research center in Cambridge, which is running the Chord clinical trial. The study looks at the use of DB-OTO gene therapy in children with mutations in the OTOF (otoferlin) gene. Through gene therapy, a functional version of this gene is supplied to patients’ inner ears.

Opal Sandy performed the surgery, which lasted just 16 minutes, on her right ear. He was also given a cochlear implant in his left ear. A few weeks later, the child could hear loud sounds, like clapping.

In statements to The Guardian, Professor Manohar Bance, surgeon and main responsible for the trial, said that this is just the beginning of gene therapies. “This marks a new era in the treatment of deafness”, highlights the specialist.

The Chord clinical trial consists of testing three children, Opal included, with the insertion of the gene into cells using a virus, in just one ear. A higher dose will be given to three other children. If deemed safe, more children could receive therapy in both ears at the same time.

A second child recently underwent gene therapy treatment at a Cambridge university hospital, with positive results. In total, 18 children from various countries will be recruited for the trial. Includes the United States, United Kingdom and Spain.

Professor Bance hopes the therapy can be applied to other more common types of hearing loss: “I hope we can start using gene therapy in young children where we actually restore their hearing and they don’t need to have cochlear implants and other technologies that need be replaced.”