First-ever gene therapy restores hearing in both ears of children born deaf
Technology
In recent years, gene therapy has emerged as a potential treatment
(Web Desk) - The world’s first clinical trial to administer gene therapy to both ears in children born with inherited deafness has been conducted.
More than 26 million people worldwide are born with congenital deafness. Unfortunately, no pharmaceutical drugs or treatments are available today to cure genetic deafness.
However, in recent years, gene therapy has emerged as a potential treatment option for restoring hearing ability and improving the quality of life for millions of people worldwide.
The clinical trial was held at the Eye & ENT Hospital of Fudan University in Shanghai, China, in collaboration with researchers from the Mass Eye and Ear Hospital in Boston, Massachusetts.
This innovative gene therapy experiment has shown promise in recovering hearing in both ears affected by OTOF gene mutations that cause autosomal deafness.
“The results from these studies are astounding,” said Zheng-Yi Chen, study co-senior author and an associate scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear.
“We continue to see the hearing ability of treated children dramatically progress and the new study shows added benefits of the gene therapy when administrated to both ears, including the ability for sound source localization and improvements in speech recognition in noisy environments,” Chen added
This genetic trial builds on the first phase trial, in which the treatment was only provided to one of the children’s ears. The trial’s results were released in January of this year.
However, the team’s main goal has been to restore hearing abilities in both ears and allow patients to perceive sounds in three dimensions.
This three-dimensional hearing is key for effective communication and essential for everyday tasks like driving.
This led to this second trial that involved five children — the first child was given bilateral (two ears) gene therapy in July 2023.
In this first-of-a-kind bilateral trial, doctors delivered gene therapy to both ears to treat DFNB9.
The therapy involves delivering functional copies of the human OTOF gene via adeno-associated virus (AAV) into the inner ear.
