by In a groundbreaking development, an 11-year-old boy has regained his hearing after undergoing a cutting-edge gene therapy. This treatment, performed at the Children’s Hospital of Philadelphia (CHOP), marks a significant milestone in the field of genetic hearing loss treatment. The success of this procedure brings hope to individuals worldwide who experience hearing loss due to genetic mutations.
Aissam Dam, the recipient of the groundbreaking gene therapy, was born profoundly deaf due to a rare gene abnormality. This genetic defect prevented the production of otoferlin, a protein essential for the hair cells in the inner ear to convert sound vibrations into chemical signals that can be relayed to the brain. Abnormalities in the otoferlin gene account for only 1-8% of congenital hearing loss cases.
Dr. John Germiller, the director of clinical research for CHOP’s otolaryngology division, expressed his excitement, stating, “Gene therapy for hearing loss is something that we physicians and scientists in the world of hearing loss have been working toward for over 20 years, and it is finally here.” He further emphasized that this breakthrough could pave the way for utilizing gene therapy to address other genetic causes of childhood hearing loss, potentially encompassing over 150 different genes.
The procedure, which took place on October 4, 2023, involved a surgical intervention that partially elevated Aissam’s eardrum. Then, a modified virus capable of carrying functional copies of the otoferlin gene was injected into the cochlea’s internal fluid. As a result, the hair cells were prompted to produce the missing protein, restoring their functionality.
Four months after the treatment, Aissam’s hearing has significantly improved. Although he still experiences mild-to-moderate hearing loss, he can now hear sounds for the first time in his life, marking a tremendous achievement. Although The New York Times reported that Aissam may not acquire language skills, as the optimal language acquisition period closes around the age of five, his ability to hear is still a remarkable outcome.
The study, sponsored by Akouos, Inc, a subsidiary of Eli Lilly and Company, was authorized by the US Food and Drug Administration (FDA). However, the FDA prioritized the research to be conducted on older children as a precautionary measure.
This landmark gene therapy trial represents just one of several endeavors taking place globally. Researchers in the United States, Europe, and China are conducting or preparing to initiate similar studies, with several other children reportedly experiencing successful outcomes.
Dr. Germiller highlighted the importance of gathering more data from patients of various ages who have undergone gene therapy to gain a better understanding of the extent to which hearing improvement can be attained and sustained over the long term. As further breakthroughs occur in this field, the potential for restoring hearing in individuals with genetic hearing loss continues to expand.
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1. Source: Coherent Market Insights, Public sources, Desk research
2. We have leveraged AI tools to mine information and compile it
