At the Children's Hospital of Philadelphia (CHOP), an 11-year-old boy from Morocco is hearing the world for the very first time thanks to a revolutionary new genetic therapy that has cured his deafness.

While the gene involved is quite rare, the milestone represents a breakthrough in the treatment of patients around the world with hearing loss caused by dozens of different genetic mutations and marks another innovative move for gene and cell therapy in a new area of medicine.

Born into a poor community in Morocco, Aissam Dam experienced a silent world, having inherited a form of hereditary deafness called otoferlin deafness. The gene otoferlin is found mutated in 200,000 people worldwide, and the mutation destroys a protein in the inner ear's hair cells necessary to transmit sound to the brain.

The New York Times reports that of the several kinds of hereditary deafness, an otoferlin mutation has been the easiest target since the hairs in the inner ear can live on for decades, while other genetic mutations cause the death of the hairs during infancy or even in the womb.

"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," said John A. Germiller PhD, an attending surgeon and Director of Clinical Research in the Division of Otolaryngology at CHOP.

Just because it was something like a low-hanging fruit, reversing or curing otoferlin was no picnic. The inner ear and the cochlea are complex, closed-off environments, and several challenges requiring years of study and testing had to be overcome.

But as for the result, young mister Aissam had some convincing words for The Times.

"There's no sound I don't like," Aissam said with the help of interpreters. "They're all good."

When asked his favorite, he signed back "people."

The experimental trial took place on October 4, 2023, when Aissam underwent a surgical procedure at CHOP where an endoscope that allows the eardrum to be partially lifted allowed for an investigational medical device to be transiently inserted into the "round window," a tiny entry point into the cochlea.

A single, small dose of a gene therapy (AK-OTOF), developed by a subsidiary of the pharma giant Eli Lilly called Akouos, and containing copies of the normal OTOF gene, was then delivered directly to the inner ear.

Now, almost four months since receiving the investigational gene therapy in one ear, he is literally hearing sound for the first time in his life. He can hear his father's voice, the sound of a car passing by, and even the scissors clipping his hair. Under healthy conditions, his hearing would be considered mildly impaired.

"As more patients at different ages are treated with this gene therapy, researchers will learn more about the degree to which hearing is improved and whether that level of hearing can be sustained over many years," Germiller said. "What we have learned from following this patient's progress will help direct our efforts toward helping as many patients as we can."

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