Children with total deafness regain hearing after “revolutionary” gene therapy: “Like a miracle”
{{#rendered}} {{/rendered}}Five children born completely deaf saw their hearing loss reversed after receiving “revolutionary” gene therapy.
The clinical trial, co-led by Mass Eye and Ear in Boston and Fudan University Eye & ENT Hospital. in Shanghaiwas the first in the world to apply gene therapy to children in both ears, according to researchers.
The research was just published in Nature Medicine on June 5.
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In addition to regaining their hearing, the children in the trial – aged 1 to 11 – were also able to identify the origin and location of sounds, even in noisy environments, the researchers said.
This was a follow-up to an earlier trial that began in December 2022, in which the research team successfully performed gene therapy in only one ear. This new study showed that treating both ears resulted in even greater benefits.
{{#rendered}} {{/rendered}}Five children born completely deaf have had their hearing loss reversed after receiving “revolutionary” gene therapy. (Fudan University Eye and ENT Hospital)
All the children in the study suffered from a hereditary form of deafness called DFNB9, caused by mutations in the OTOF gene.
This disease occurs when the OTOF gene is unable to produce a protein called otoferlin, which is essential for transmitting sound signals from the ear to the brain.
As a result, the children could neither hear nor speak.
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“Children were chosen because they would benefit most from early gene therapy intervention, particularly in speech acquisition,” said study author Zheng-Yi Chen, DPhil, a researcher. associated with Eaton-Peabody Laboratories at Mass Eye and Ear in Boston. Fox News Digital in an interview.
“But from a safety standpoint, it’s riskier for kids.”
{{#rendered}} {{/rendered}}How the procedure works
During the “minimally invasive” surgical procedure, doctors administered an injection of the human OTOF gene into the inner ears of children.
The children remained in the hospital for approximately seven to ten days for observation.
“After four weeks, the children showed auditory perception in tests, and then gradually acquired the ability to speak,” Chen said.
{{#rendered}} {{/rendered}}Dr. Yilai Shu is shown communicating with a young patient at Fudan University Eye and ENT Hospital. (Fudan University Eye and ENT Hospital)
Within families, response to sound was noted after two to three weeks.
“All five patients regained their hearing, improved their speech perception and their perception of sound sources in noisy environments,” Chen said.
Participants experienced only mild side effects, such as fever and vomiting.
{{#rendered}} {{/rendered}}“This is the first time in history that hearing loss has been reversed using gene therapy.”
“There were no serious adverse effects,” he said. “They all recovered without any intervention.”
Gene therapy aims to be a single treatment and will not need to be repeated, the researchers said, although children will likely need speech therapy.
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{{#rendered}} {{/rendered}}So far, there is no single treatment for hearing loss other than cochlear implants, according to researcher Yilai Shu MD, PhD, director of the Genetic Hearing Loss Diagnosis and Treatment Center at Fudan Hospital. from Shanghai.
“This is the first time in history that hearing loss has been reversed through gene therapy,” Shu told Fox News Digital. “And of course we think it will have a profound impact on children’s lives.”
Dr. Yilai Shu’s team works in the laboratory of Fudan University Eye and ENT Hospital. (Fudan University Eye and ENT Hospital)
Chang Yiyi, a Shanghai mother whose 3-year-old son, Zhu Yangyang, participated in the trial, spoke to Fox News Digital about the experience.
{{#rendered}} {{/rendered}}“When Zhu couldn’t speak at age 2 and didn’t respond to sounds, we realized there was a problem,” she said.
After hearing tests, it was determined that Yiyi’s son suffered from total deafness.
“It was incredible, the best feeling. It was like a miracle.”
“He was very frustrated because he couldn’t understand, couldn’t speak, couldn’t hear,” she said.
{{#rendered}} {{/rendered}}Twenty-three days after receiving the gene therapy, the boy first responded to someone calling him.
“It was amazing – the best feeling,” Yiyi told Fox News Digital. “It was like a miracle“.
“Now he can say ‘Mommy’ and ‘I want’ and some simple sentences.”
{{#rendered}} {{/rendered}}Dr. Yilai Shu (center) is pictured in the operating room of Fudan University Eye and ENT Hospital. (Fudan University Eye and ENT Hospital)
About 430 million people worldwide suffer from disabling hearing loss, including 34 million children, according to the study. World Health Organization.
More than 50% of cases of hearing loss are genetic in origin.
Gene therapy shows promise but there are limits, expert says
Dr. Amy Sarow, the Based in Michigan The senior audiologist at Soundly, a hearing healthcare marketplace, noted that gene therapy has seen some success in treating cancer and eye diseases, as well as other emerging areas.
{{#rendered}} {{/rendered}}“It’s exciting to think about how gene therapy could impact millions of hearing-impaired people around the world,” Sarow, who was not involved in experimental gene therapy, told Fox News Digital.
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“However, it is essential to emphasize that there are many causes of hearing loss and one type of gene therapy will not suit all types.”
{{#rendered}} {{/rendered}}Even among genetic causes In deafness, different genes can cause abnormalities or dysfunction affecting different hearing pathways, according to Sarow.
“Thus, the development of specific therapeutic interventions depends on causality and will still take time to develop.”
Dr. Yilai Shu examines a young patient at Fudan University Eye and ENT Hospital. (Fudan University Eye and ENT Hospital)
Additionally, Sarow noted, a “reversal” of hearing loss does not mean that an individual will fully regain their normal hearing ability.
{{#rendered}} {{/rendered}}“The first three years of life are very important for language acquisition, and even though these children would be behind their normally hearing peers (having spent the first years of their life profoundly deaf), they would still have the opportunity to “catch up to some extent,” she said.
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“Research tells us that the younger the intervention, the better the potential language development.”
{{#rendered}} {{/rendered}}As with any intervention, gene therapy can carry risks. “A potential risk is that the treatment may not be successful in all cases,” Sarow said.
“Another potential risk is that the targeted gene therapy may not work in the targeted region.”
And after?
The next step is to follow trial patients for a longer period of time to ensure the stability of positive results, Shu said.
{{#rendered}} {{/rendered}}Until now, there was no single treatment for hearing loss other than cochlear implants, according to one of the researchers. (iStock)
Based on the results of the first study, researchers expect that patients’ hearing abilities will continue to improve over time.
“Next, we want to expand the treatment to older patients and evaluate the effectiveness of the treatment for aging adults,” he said.
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{{#rendered}} {{/rendered}}“Ultimately, we want the patient to have a choice as to which treatment option they want to pursue.”
The researchers also plan to begin the process of obtaining FDA approval to bring the gene therapy to the United States.
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{{#rendered}} {{/rendered}}“We are working to make this available to people outside of China, including in the United States, as quickly as possible,” Shu told Fox News Digital.
Researchers also hope to expand this type of gene therapy to other types of deafness in the future.
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