Five children who were born completely deaf have had some of their hearing loss reversed after receiving a “groundbreaking” gene therapy.
The clinical trial was jointly led by Massachusetts Eye and Otolaryngology in Boston and the Fudan University Hospital of Eye and Otolaryngology. ShanghaiAccording to the researchers, this is the first time that the team has administered gene therapy to children in both ears.
The study was just published in Nature Medicine on June 5th.
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The children, aged between one and 11, not only recovered their hearing but also were able to identify the source and location of sounds in noisy environments, according to the researchers.
This is a follow-up to a previous trial that began in December 2022, in which the research team successfully administered the gene therapy in just one ear. The new study shows that treating both ears could produce even greater benefits.
Five children who were born completely deaf have had their hearing restored after receiving a “groundbreaking” gene therapy. (Fudan University Eye and ENT Hospital)
All of Children who participated in the study The patient had a genetic form of hearing loss called DFNB9, which is caused by a mutation in the OTOF gene.
The condition occurs when the OTOF gene fails to produce a protein called otoferin, which is essential for transmitting sound signals from the ear to the brain.
As a result, the children could no longer hear or speak.
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“These children were selected because they are thought to benefit most from early intervention with gene therapy, particularly in language acquisition,” study author Dr. Chen-Yi Chen, an associate scientist at the Massachusetts Eye and Ear Institute of Eaton Peabody in Boston, said in an interview with Fox News Digital.
“But from a safety standpoint, it’s more dangerous for children.”
How the procedure works
During a “minimally invasive” surgical procedure, doctors transfected the human OTOF gene Children’s inner ear.
The children were hospitalized for observation for approximately seven to 10 days.
“After four weeks, the children were tested to recognize hearing and then gradually gained the ability to speak,” Chen said.
Dr. Yilai Shu communicates with a young patient at the Eye and ENT Hospital of Fudan University. (Fudan University Eye and ENT Hospital)
Within families, reactions to sound were observed within 2-3 weeks.
“All five patients experienced restored hearing, improved speech perception, and improved perception of sound sources in noisy environments,” Chen said.
Participants experienced only mild side effects, such as fever and vomiting.
“This is the first time in history that hearing loss has been reversed through gene therapy.”
“There were no serious side effects,” he said. “They all recovered without any intervention.”
Gene therapy is One-time treatment The researchers said the children would likely need speech therapy but would not need to be retested.
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Until now, there has been no standalone treatment for hearing loss other than cochlear implants, said researcher Yilai Shu, MD, director of the Hereditary Hearing Loss Diagnostic and Treatment Center at Fudan Hospital in Shanghai.
“This is the first time in history that hearing loss has been reversed through gene therapy,” Hsu told Fox News Digital, “and of course, we believe this is going to have a profound impact on children’s lives.”
Pictured is Dr Yilai Shu’s team working in his lab at the Eye and ENT Hospital of Fudan University. (Fudan University Eye and ENT Hospital)
Zhang Yiyi, a mother in Shanghai, spoke to Fox News Digital about her 3-year-old son Zhu Yanyang’s experience taking part in a clinical trial.
“We realised we had a problem when Chu was two years old and still not speaking or responding to sounds,” she said.
A hearing test revealed that Yiyi’s son was totally hearing impaired.
“It was unbelievable, it was the best feeling. It was like a miracle.”
“It was very frustrating because he couldn’t understand, he couldn’t talk, he couldn’t hear,” she said.
Twenty-three days after receiving the gene therapy, the boy responded for the first time to someone calling out to him.
“It was unbelievable, it felt amazing,” Yee told Fox News Digital. Like a miracle“
“Now he can say ‘mama’ and ‘I want’ and speak simple sentences.”
Pictured is Dr. Yilai Shu (center) in the operating room at Fudan University Eye and ENT Hospital. (Fudan University Eye and ENT Hospital)
Approximately 430 million people worldwide are hearing impaired, of which 34 million are children. who.
Over 50% of hearing loss is genetic.
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Dr. Amy Sarrow Based in Michigan The lead audiologist at hearing healthcare marketplace Soundly noted that gene therapy has seen some success in treating cancer and eye diseases, as well as other emerging fields.
“It’s exciting to think about how gene therapy could impact the millions of people with hearing loss around the world,” Thurow, who is not involved in the experimental gene therapy, told Fox News Digital.
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“However, it is important to emphasize that the causes of hearing loss are diverse and no one gene therapy is suitable for all types of hearing loss.”
Among them Genetic causes In the case of hearing loss, different genes can cause abnormalities or dysfunctions that affect different hearing pathways, Thurow said.
“The development of specific therapeutic interventions is therefore dependent on causality and will likely take some time to develop.”
Dr. Xu Yilai examines a young patient at the Eye and ENT Hospital of Fudan University. (Fudan University Eye and ENT Hospital)
Additionally, Thurow pointed out that “recovery” of hearing loss does not necessarily mean that normal hearing is fully restored.
“The first three years of life are crucial for language acquisition, and although these children will fall behind their normal-hearing peers (because they have spent the first few years of life with profound hearing loss), they still have the potential to ‘catch up’ to some extent,” she said.
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“Research has shown that the younger the age of intervention, the more beneficial the impact on underlying language development.”
As with any therapeutic intervention, gene therapy comes with risks. “One potential risk is that the treatment may not necessarily be successful,” Thurow said.
“Another potential risk is that targeted gene therapy may not be effective in the targeted area.”
What’s next?
The next step is to Clinical trial patients Xu said trials would continue for a longer period to ensure the positive results remained stable.
According to one of the researchers, until now there was no treatment for hearing loss other than cochlear implants. (iStock)
Based on the results of the initial study, the researchers expect patients’ hearing to continue to improve over time.
“We would then like to expand the study to include older patients and evaluate the effectiveness of the treatment in older people,” he said.
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“Ultimately, we want patients to have the choice of which treatment they choose.”
The researchers also plan to begin the process of seeking FDA approval to bring the gene therapy to the United States.
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“We are working to get this to people outside of China, including in the United States, as quickly as possible,” Fox News Digital reported.
The researchers hope to eventually apply this type of gene therapy to treat other types of hearing loss.
