Is Gene Therapy the Future for Restoring Children’s Hearing?

The medical community has been trialling gene therapy as a treatment for genetic deafness since 2019. Last year, a deaf British toddler named Opal received an infusion of corrected genes into her inner ear. Her parents noticed changes in her hearing within 4 weeks, and obvious improvements within twenty-four. Our team of pediatric audiologist at Soundwave Hearing Care in Calgary find this exciting and want to share more information with you about the promising future of gene therapy as a way of restoring children’s hearing.  

Infant Deafness 

Approximately two thousand Canadian children are born with hearing impairment each year. These range from moderate bilateral hearing loss to profound loss. For 50 to 60 percent of children born with hearing loss, genetics is the cause. 

Genetic Hearing Loss 

Various genes can mutate and cause infant hearing loss, but the one currently receiving attention the OTOF gene. It makes a protein that facilitates communication between cells in the ear and the hearing nerve.  

Opal’s Treatment 

Young Opal, mentioned above, and two other children received an infusion of corrected OTOF genes into one of their inner ears through a catheter during a surgery that resembles fitting a cochlear implant, which Opal previously had. For the study, Opal’s implant was removed, and she received the genetic infusion for 16 minutes.  

Within a month, her parents noticed changes in Opal’s hearing, and after 6 months, she was reacting to sounds she previously had never noticed without her cochlear implant. Opal’s audiologists consider her to have normal hearing. 

Next Phases of the Trial 

Another trio of deaf children will receive the same single-ear treatment but at a higher dose. If the study progresses, a third group of children will receive simultaneous doses in both ears. The total of 18 children, from the UK, US, and Spain, will be followed by the researchers and receive hearing tests for 5 years.    

Other Trials 

An 11-year-old Italian boy received OTOF gene therapy the same year as Opal. Though part of a different trial, the boy’s experience was similar. The pre-teen went from complete hearing loss to what audiologists call mild hearing loss. 

Five deaf children aged 1 to 11 also received OTOF gene therapy in a Chinese trial in 2024. They could locate sounds, recognize speech, and dance to music within weeks of receiving infusions in both ears.  

Where Do We Go From Here? 

It is difficult to predict the future of ongoing trials, but Opal’s experience and those of her Chinese and Italian counterparts are encouraging for people with an OTOF gene mutation. It is for researchers to apply the process to other mutations and continue to find effective hearing restoration treatments.  

Non-genetic Infant Hearing Loss 

If your child is experiencing hearing loss, contact Soundwave Hearing Care in Calgary, Grande Prairie, or High River to book an appointment for a pediatric hearing test with our team of audiologists and trained hearing professionals. We offer complete pediatric hearing care in Calgary and around Alberta.