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Aissam Dam was born “profoundly deaf” due to a very rare abnormality in a single gene.
“Gene therapy for hearing loss is something that doctors and scientists in the hearing loss world have been working on for more than 20 years, and it’s finally here,” said surgeon John Germiller, director of clinical research at the division of otolaryngology at CHOP.
“While the gene therapy we performed on our patient was to correct an abnormality in a very rare gene, these studies may open the door for future use of some of the more than 150 genes that cause childhood hearing loss.”
In patients like Aissam, a defective gene prevents the production of otoferlin, a protein necessary so that the “hair cells” of the inner ear can convert sound vibrations into chemical signals that are sent to the brain. Otoferlin gene defects are very rare and account for between 1 and 8 percent of hearing loss present from birth.
On October 4, 2023, he underwent a surgical procedure that involved partially lifting his eardrum and then injecting a harmless virus, which had been modified to carry functional copies of the otoferlin gene, into the internal fluid of his cochlea. As a result, the hair cells began to produce the missing protein and function properly.
Nearly four months since receiving treatment in one ear, Aissam’s hearing has improved to the point that he only has mild to moderate hearing loss and is “literally hearing a sound for the first time in his life,” according to the statement. .
The New York Times reported that despite being able to hear, Aissam, who was born in Morocco and later moved to Spain, may never learn to speak, as the brain’s window for acquiring speech closes around age five. . The U.S. Food and Drug Administration, which gave the green light to the study, wanted to begin the research with older children first, for safety reasons.
The trial, sponsored by Akouos, Inc, a wholly owned subsidiary of Eli Lilly and Company, is one of several underway or about to begin in the United States, Europe and China, where a handful of other children have already been cured.
“As more patients of different ages are treated with this gene therapy, researchers will learn more about the degree to which hearing improves and whether that level of hearing can be maintained for many years,” Germiller said.
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