Trials of gene therapy help deaf infants regain hearing

Success in a gene therapy trial has restored hearing to deaf children.

Six children with a gene mutation that impacts ear-brain signal transmission were treated in the experiment, which was carried out in Fudan, China, at the Eye & ENT Hospital of Fudan University and in collaboration with a group from the Massachusetts Eye and Ear Infirmary and Harvard Medical School.

In 2022, researchers studied the otoferlin gene in a 26-week experiment with children aged one to seven. The research indicated that five out of six of the participants had hearing gains or improvements. Children who had hearing loss also developed a better perception of spoken language.

The three older children in the 26-week research who had their cochlear implants deactivated were able to comprehend and react to spoken language after the trial. According to the Harvard Gazette, two of them managed to carry on a phone call while being able to identify speech despite the background noise.

Early trial findings show treatment was effective, according to the Children’s Hospital of Philadelphia, which released the news on Tuesday.

“Although the gene in question is extremely uncommon, this significant achievement signifies a major stride forward in the field of gene and cell therapy, opening up new avenues of treatment for individuals globally who suffer from hearing loss due to a myriad of genetic abnormalities,” the hospital announced.

At birth, the Philadelphia infant had hearing loss caused by the otoferlin gene. He or she was eleven years old. He was born with significant hearing loss in both ears. He had surgery on October 4, 2023, and since then, his hearing has significantly improved to the point that he “only had mild to moderate hearing loss in the ear that was treated.”

Using a device that partially elevates the eardrum, the gene therapy was inserted into the inner ear during the surgical operation that the youngsters had. The Philadelphia boy was administered a single, tiny dose containing copies of the normal gene.

The hospital reported that approximately one in five hundred infants are born with a hereditary hearing impairment and that more than 150 genes have been linked to the condition.