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An 11-year-old youngster receives breakthrough gene therapy and hears for the first time

Eleven-year-old Aissam Dam was raised in a world of extreme quiet. He had never heard anything because he was born deaf. Living in a low-income neighborhood of Morocco, he was illiterate and used a sign language he created to communicate. Aissam’s family brought him to a hearing expert last year when they moved to Spain, and the doctor gave him an unexpected recommendation: Aissam could be qualified for a gene therapy clinical study.

Aissam received treatment at Children’s Hospital of Philadelphia on October 4, making history as the first individual in the US to get gene therapy for congenital deafness. The intention was to give him hearing, but neither the treatment’s efficacy nor the extent to which it would operate was known to the researchers. The toddler who had never heard of sound was exposed to a new universe thanks to the successful therapy. During an interview last week, Aissam said, “There’s no sound I don’t like,” with the assistance of translators. “They’re all good.”
Assam is one of the millions of individuals who are born deaf, but there are hundreds of millions of others who suffer from hearing loss that is considered incapacitating. This very unusual type is brought about by a mutation in the otoferlin gene. Worldwide, around 200,000 persons suffer from otoferlin deafness. The purpose of gene therapy is to implant a functioning gene in the ears of patients in lieu of the mutant otoferlin gene. The success of individuals like Aissam, according to experts, may pave the way for gene treatments that target other types of congenital deafness, even if it will take years for physicians to enroll many more patients—and younger ones—to further evaluate the therapy. Dr. Dylan Chan, a pediatric otolaryngologist who was not part in the experiment, called it a “groundbreaking” study.
The one in which Aissam took part is backed by Eli Lilly and Akouos, a little biotechnology company they control. Of the five trials that are either in progress or scheduled to begin, Aissam’s is the only one that is not in China or Europe.
Aissam’s condition is caused by a gene abnormality that inhibits the creation of otoferlin, a protein required by the inner ear’s “hair cells” to transform sound waves into chemical signals that are sent to the brain. A harmless virus with new otoferlin genes is used in gene therapy, and two drops of liquid are carefully administered down the length of the cochlea to deliver the genes to each hair cell. Despite the progress, Dr. John Germiller, the otolaryngologist who oversaw the study, said that the experts acknowledge that Aissam could never be able to comprehend or speak a language. According to him, the brain’s window for learning to speak shuts at the age of five. However, Aissam did not hesitate to answer when asked whether there was a sound that he especially enjoyed. He said, “People,” in sign language.

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