A GROUNDBREAKING gene therapy has enabled congenital deafness patients to hear everyday conversations, according to a new study published in Nature. The research marks a major milestone in the fight against hereditary hearing loss, offering new hope to millions worldwide. The nationwide multicenter clinical trial, led by Shanghai’s Eye and ENT Hospital of Fudan University, involved 42 patients aged 9 months to 32 years. After receiving just a single injection and completing a 2.5-year follow-up, every patient in the study regained hearing sufficient to understand normal conversations. The results are remarkable: 57% could hear the soft sound of pages turning in a quiet library, and 43% could detect whispers. Globally, this is the earliest, largest, and longest-followed clinical study in the field of gene therapy for deafness. It also includes the youngest and oldest patients ever reported in such research. The therapy specifically targets DFNB9 deafness, a severe form caused by mutations in the OTOF gene. This condition typically results in profound bilateral hearing loss from birth, significantly affecting language acquisition and cognitive development. Among the estimated 26 million people worldwide living with congenital deafness, those with the DFNB9 type face particularly serious challenges. The latest multicenter study aimed to reveal long-term patterns of hearing and speech improvement while identifying key factors influencing treatment effectiveness. The findings show that younger patients — especially those aged 5 months to 3 years — experienced more significant improvements than adults. Additionally, patients who showed certain pre-treatment responses tended to have better hearing recovery. Adults also benefited. Zhang Wenxi, 32, received the treatment in 2024. She had congenital profound deafness and had received a cochlear implant at age 6. “On the second morning after receiving the gene therapy, it was the first time in my life that I heard the sound of running water from a faucet,” she said. Her hearing improved from detecting sounds at 100 decibels before treatment to about 55 decibels now. “It is truly an incredible journey of rebirth.” (SD-Agencies) |