YOUTH with congenital deafness treated with gene therapy showed better progress on certain hearing measures than counterparts treated with cochlear implantation, a cohort study in China indicated.

The 11 gene therapy recipients studied showed stable hearing recovery and faster improvements in auditory and speech performance compared with the 61 cochlear implantation recipients, reported researchers led by doctor Shu Yilai of the Eye and Ear, Nose, and Throat Hospital of Fudan University in Shanghai.

And participants who received gene therapy also outperformed those who received cochlear implantation in speech-in-noise recognition and music perception, the group noted in JAMA Neurology.

The study, which was not blinded, analyzed outcomes for 1- to 18-year-olds who received gene therapy or cochlear implantation or both for congenital severe to complete hearing loss at a tertiary hospital in China from December 2022 to November 2024.

The 72 children were evaluated across multiple dimensions, such as hearing thresholds, speech recognition, and music perception, one year after receiving treatment.

This was the first global study systematically comparing multidimensional auditory and speech perception levels of children who received cochlear implants or OTOF gene therapy, which targets the otoferlin protein crucial for hearing.

There are no clinical drugs for congenital deafness, with cochlear implants being the only option available so far. However, they are unlikely to restore natural hearing, with limited noise and music perception improvements, and require long-term maintenance.

This Chinese study represents a huge breakthrough in the treatment of congenital deafness. The OTOF gene therapy is expected to enter clinical use soon, prompting a transformation in the approach to deafness treatment and providing a reference for treating other types of hearing loss.

About 1/5 of the global population suffers from hearing loss, and about 26 million people have congenital deafness, 60% of whom are related to genetics. More than 200 known pathogenic genes have been identified.

(SD-Agencies)