Five children, born completely deaf, experience a reversal of hearing loss following a “groundbreaking” gene therapy conducted in a clinical trial co-led by Mass Eye and Ear in Boston and the Eye & ENT Hospital of Fudan University in Shanghai. Published in Nature Medicine on June 5, the trial marks the first application of gene therapy to children in both ears globally.
Ages ranging from 1 to 11 years old, the children not only regain their hearing but also demonstrate the ability to discern sounds’ origins and locations, even in noisy settings. Building on a previous trial from December 2022, where gene therapy was administered in just one ear, this new study reveals enhanced benefits with treatment in both ears.
All participants had hereditary deafness known as DFNB9, stemming from mutations in the OTOF gene. This gene’s inability to produce otoferlin protein disrupts sound signal transmission from the ear to the brain, resulting in hearing and speech impairments.
The selection of these children underscores the potential of early intervention through gene therapy, especially for speech development. Yet, the procedure carries inherent risks, particularly for pediatric patients.
In the minimally invasive surgery, doctors inject the human OTOF gene into the inner ears of the children. Following the procedure, the children undergo observation in the hospital for approximately seven to ten days. Within four weeks, they exhibit hearing perception improvements in tests, gradually regaining speech capabilities.
