Gene therapy: Several deaf children can now hear after new treatment

A new gene therapy offers hope for curing deafness. Yiyi, a Chinese girl, was born completely deaf. She participated in a gene therapy trial and regained hearing in the treated ear about a month after the treatment. In a video, her mother Qin Lixue demonstrates her daughter's improved hearing abilities. Qin Lixue holds her hands in front of her mouth when speaking to prevent Yiyi from lip-reading:

Clouds, one by one, blossomed in the mountains.

Yiyi then repeats:

Clouds, one by one, blossomed in the mountains.

During the procedure, physicians utilized a virus to introduce replacement DNA to the cells located in Yiyi's inner ear responsible for detecting vibrations. This allows for proper sound transmission to her brain. Yilai Shu, a surgeon and scientist at Fudan University in Shanghai and the experiment's lead, provides clarification.

We were careful, and a little bit nervous, because it was the first in the world.

A big step

In December, Shu and his team conducted the initial treatments on test subjects. Besides Yiyi, nine other deaf children received treatment using the new procedure as part of the study. Chinese scientists claim that these are the world's first individuals to recover their natural hearing. Zheng-Yi Chen, a professor at Mass Eye and Ear, a hospital affiliated with Harvard University in Boston, who was involved in planning and conducting the study, explains:

Before the treatment, if you put them in a movie theater with the loudest sound, they wouldn’t hear it. Now they can hear close to normal speech, and one can hear a whisper.

On average, patients have regained approximately 60-65% of their hearing ability, enabling them to hear regular conversations and louder sounds such as a motorcycle. Shu presented the results of the first five treated cases at a meeting of the European Society for Gene and Cell Therapy, with only four patients successfully regaining their hearing.

Due to their young age, some children receiving the therapy cannot share their experiences verbally. However, there have been noticeable behavioral changes, such as one toddler beginning to say words like "mama" and "baba" shortly after undergoing therapy.

Hope for affordable treatment

The new treatment is exclusively for deaf individuals with a genetic defect in the protein otoferlin, which causes complete deafness from birth. This defect is rare, affecting only one to three percent of those deaf since birth. The study was sponsored by Shanghai Refreshgene Therapeutics, a small biotech company. The primary goal of commercialization, according to founder Nova Liu, is to make the therapy affordable.