This week marks a turning point for hereditary deafness. On Wednesday, a new study reported the results of a clinical trial showing improvements in hearing recovery and speech recognition in five out of six children who received the hearing loss gene**. No dose-limiting toxicities were reported. The trial, which began in December 2022 and was conducted in China (in collaboration with Mass Eye and Ear), was the first to genetically ** DFNB9 in children with hereditary deafness. This study represents the first human clinical trial using the gene *** for this disease.
Earlier this week, Akouos (a wholly owned subsidiary of Eli Lilly and Company) announced positive preliminary clinical results from its II II AK-OTOF-101 gene** study. The first participant to receive the gene** was an 11-year-old child with severe hearing loss from birth who had hearing recovery within 30 days of AK-OTOF administration.
Both groups plan to present more data on these studies at the ENT Research Association's annual meeting in the first week of February.
If children can't hear, their brains can develop abnormally without intervention," said Dr. Zheng-Yi Chen, an associate scientist at Mass Eye and Ear's Eaton-Peabody Lab and associate professor of otolaryngology-head and neck surgery at Harvard Medical School. "The results of this study are truly remarkable. We have seen significant improvements in children's hearing every week, and their ability to speak has also been restored.
The findings were published in The Lancet, "A**1-Hotof Gene**Autosomal Recessive Deafness 9: A One-Arm Test".
More than 1.5 billion people worldwide suffer from hearing loss, of which about 26 million are born with deafness. More than 60% of hearing loss in children is due to genetic causes. DFNB9 is an inherited disorder caused by mutations in the OTOF gene and failure to produce functional otoferrin, which is necessary to carry sound signals from the ear to the brain. There are currently no FDA-approved drugs to help** hereditary deafness.
The Lancet study describes a trial in six children with DFNB9, a specific form of autosomal recessive hearing loss caused by mutations in the OTOF (otoferrin) gene. The participants were observed at the Otolaryngology Hospital of Fudan University for 26 weeks. Collaborators at Mass Eye and Ear utilized adeno-associated virus (A**), which carries the human OTOF gene, to introduce the gene into the patient's inner ear through a special surgical procedure. A single injection of the viral vector is used in different doses.
All six children in the study were completely deaf with an average auditory brainstem response (ABR) threshold of more than 95 decibels. After 26 weeks, five children showed hearing recovery, a 40-57 dB reduction in ABR testing, a significant improvement in speech perception, and a return to the ability to conduct normal conversations. At follow-up of patients, 48 adverse events were observed, of which the vast majority (96%) were low-grade and the rest were transient with no long-term effects.
This study provides evidence of the safety and efficacy of the gene ***dfnb9, as well as their potential for other forms of hereditary hearing loss. In addition, these results contribute to the understanding of the safety of a** insertion into the human inner ear. With regard to the use of A**, the success of the double A** vector carrying two OTOF gene fragments is noteworthy. Typically, A** has a gene size limit, so for genes like OTOF that that limit, the achievement of the double viral vector opens the door for A** to be used with other large genes that are often too large for the vector.
We were the first to initiate a clinical trial of the OTOF gene**. It's exciting that our team is translating the work from basic research in animal models of DFNB9 to hearing recovery in children with DFNB9," said Yilai Shu, MD, from the Otolaryngology Hospital of Fudan University. Shu previously worked as a postdoctoral researcher in Chen's lab at Mass Eye and Ear. "I'm very excited about our future work on other forms of hereditary hearing loss in order to make it available to more patients.
The researchers plan to expand the trial to a larger sample size and track their results for a longer period of time.
Since the invention of cochlear implants 60 years ago, there has been no effective way to deaf,**," Chen said. "This is a huge milestone and symbolizes a new era in the fight against all types of hearing loss.
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