Gene therapy has successfully restored hearing in children born with inherited deafness. A recently published small study, conducted in China, revealed significant improvements in the hearing of five out of six children who underwent experimental gene therapy. The Children’s Hospital of Philadelphia also reported similar advancements in an 11-year-old boy, while earlier this month, Chinese researchers published a study demonstrating comparable results in two other children.
Currently, the experimental therapies focus on addressing a specific rare condition. However, scientists anticipate that similar treatments could eventually aid numerous children with various types of deafness caused by genetic factors. Globally, an estimated 34 million children experience deafness or hearing loss, with genetics contributing to up to 60% of cases. Hereditary deafness has become the latest target for gene therapy, already approved for treating conditions such as sickle cell disease and severe hemophilia.
Zheng-Yi Chen of Boston’s Mass Eye and Ear, a senior author of the study published in the Lancet on Wednesday, expressed excitement about the results: “No treatment could reverse hearing loss… That’s why we were always trying to develop a therapy.” Videos documenting the patients’ progress were captured by the research team. One video shows a baby, previously unable to hear, responding to a doctor’s words six weeks after treatment. Another depicts a young girl repeating words like father, mother, grandmother, sister, and “I love you” 13 weeks after treatment.
All the children in the experiments had a condition accounting for 2% to 8% of inherited deafness, caused by mutations in a gene responsible for an inner ear protein called otoferlin. The one-time therapy delivers a functional copy of that gene to the inner ear during a surgical procedure. The study, involving six children, took place at Fudan University in Shanghai, co-led by Dr. Yilai Shu. The therapy resulted in improved hearing in five out of six children, with no major side effects reported.
While the preliminary results from other research, including studies sponsored by Regeneron Pharmaceuticals and Eli Lilly’s subsidiary Akouos, have shown positive outcomes, questions remain regarding the long-term effectiveness of these therapies and the potential for continued improvement in the children’s hearing.
Despite the promising results, some ethical concerns surround gene therapy for deafness. Teresa Blankmeyer Burke, a deaf philosophy professor and bioethicist at Gallaudet University, emphasized the importance of engaging with the deaf community about the prioritization of gene therapy, considering its potential impact on the flourishing of signing Deaf communities.
Researchers remain optimistic about the progress of their work, with Chen stating, “This is real proof showing gene therapy is working. It opens up the whole field.” The development holds significant promise for the future treatment of various forms of genetic deafness in children.
Gary P Hernal started college at UP Diliman and received his BA in Economics from San Sebastian College, Manila, and Masters in Information Systems Management from Keller Graduate School of Management of DeVry University in Oak Brook, IL. He has 25 years of copy editing and management experience at Thomson West, a subsidiary of Thomson Reuters.
