- Children with hereditary deafness have regained their hearing through a groundbreaking gene therapy trial conducted in China.
- The trial involved injecting a functional version of the OTOF gene into the inner ear of six children with DFNB9, a genetic form of deafness caused by mutations in the OTOF gene.
- Five out of the six children regained their hearing and the ability to have normal conversations, marking a significant milestone in the field of hearing loss treatment.
Additional Coverage:
- Children with genetic deafness have hearing restored with gene therapy, study finds (abcnews.go.com)
A new study published on Wednesday revealed that children with hereditary deafness have regained their hearing through gene therapy. The clinical trial, co-led by investigators from Mass Eye and Ear, focused on six children with a genetic form of deafness known as DFNB9. This type of deafness is caused by mutations in the OTOF gene, which prevents the production of otoferlin, a protein necessary for transmitting sound signals from the ear to the brain.
The trial took place at the Eye & ENT Hospital of Fudan University in Shanghai and involved injecting an inactive virus carrying a functional version of the OTOF gene into the inner ear of the children at varying doses. The participants were then observed for 26 weeks. Results published in The Lancet showed that five out of the six children, who were considered completely deaf, regained their hearing and the ability to have normal conversations.
This gene therapy trial is the first of its kind to be conducted in humans for the treatment of this specific condition and has the most patients treated and the longest follow-up period. Dr. Zheng-Yi Chen, an associate scientist at Mass Eye and Ear, expressed excitement over this milestone and noted that it will have a significant impact on the field of hearing loss treatment.
Hearing loss affects a large number of people, and until now, there hasn’t been an FDA-approved drug to treat any type of hearing loss. Dr. Chen emphasized the importance of communication in children’s development and how their brain development can be severely affected if they cannot hear or communicate, especially in their early years.
The next steps for the researchers involve expanding the study to include more patients for a larger clinical trial and monitoring them for a longer period to ensure the efficacy of the treatment. Their hope is to expand this gene therapy approach to treat other forms of genetic deafness as well.
The findings from this clinical trial will be presented at the Association for Research in Otolaryngology Annual Meeting in February. This news follows a report about the successful gene therapy treatment of an 11-year-old boy from Spain with a genetic form of hearing loss at the Children’s Hospital of Philadelphia, using a gene therapy developed by Eli Lully pharmaceutical company.