Breakthrough Gene Treatment Restores Hearing for Deaf Boy

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WASHINGTON, D.C.: In a groundbreaking development, an 11-year-old boy is experiencing the sensation of hearing for the first time in his life after receiving a revolutionary gene therapy. The Children’s Hospital of Philadelphia (CHOP), which conducted the treatment, announced this significant milestone, offering hope to individuals worldwide who suffer from hearing loss caused by genetic mutations.

Aissam Dam, born with profound deafness due to a highly rare abnormality in a single gene, can now hear thanks to this pioneering gene therapy. Surgeon 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. While the gene therapy we performed in our patient was to correct an abnormality in one very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss.”

In individuals like Aissam, a faulty gene hinders the production of otoferlin, a crucial protein necessary for the hair cells in the inner ear to convert sound vibrations into chemical signals sent to the brain. Otoferlin gene defects are extremely rare, accounting for only 1-8 percent of hearing loss present from birth.

On October 4, 2023, Aissam underwent a surgical procedure that involved partially lifting his eardrum and injecting a harmless virus, modified to carry functional copies of the otoferlin gene, into the fluid of his cochlea. As a result, the hair cells began producing the missing protein and regained their proper functionality.

After nearly four months since the treatment, Aissam’s hearing has significantly improved, with only mild-to-moderate hearing loss remaining. This achievement marks a remarkable milestone for Aissam, who can now “literally hear sound for the first time in his life,” according to the statement released by CHOP.

While this breakthrough is undoubtedly a cause for celebration, it is important to note that Aissam may face challenges in acquiring speech. The New York Times reported that due to the brain’s window for acquiring speech closing around the age of five, Aissam, who was born in Morocco and later moved to Spain, may never learn to talk. The US Food and Drug Administration, which approved the study, prioritized starting the research with older children for safety reasons.

The trial, sponsored by Akouos Inc., a subsidiary of Eli Lilly and Co., is one of several ongoing or upcoming studies in the United States, Europe, and China. In China, a handful of other children have already reportedly been cured using similar gene therapy techniques.

Germiller emphasized the importance of continued research, stating, “As more patients at different ages are treated with this gene therapy, researchers will learn more about the extent to which hearing can be improved and whether this level of hearing can be sustained over many years.”

This groundbreaking gene therapy not only offers hope to individuals like Aissam but also paves the way for future advancements in treating childhood hearing loss caused by genetic mutations. The medical community is optimistic about the potential of gene therapy to transform the lives of countless individuals worldwide, providing them with the gift of hearing.

Source: The Manila Times

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