An 11-year-old boy is able to hear for the very first time in his life thanks to a groundbreaking gene therapy procedure, according to the Children's Hospital of Philadelphia (CHOP).
The boy -- who was born profoundly deaf in both ears due to hereditary hearing loss -- was the first person to receive the experimental gene therapy treatment, CHOP said.
“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, MD, PhD, an attending surgeon and Director of Clinical Research in the Division of Otolaryngology at CHOP, and Associate Professor at Perelman School of Medicine at the University of Pennsylvania. “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.”
The boy underwent a procedure on Oct. 4, 2023, in which the gene therapy was placed into his inner ear using an endoscope. The endoscope allowed the ear drum to be partially lifted so that a medical device was inserted into a tiny entry point into the cochlea known as the "round window." A single, small dose of the gene therapy was then delivered directly to his inner ear.
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The procedure was the first time a gene therapy has been used as a potential treatment for hereditary hearing loss in the United States, CHOP said.
Four months after the procedure, the boy can now hear sound for the first time in his life, including his father's voice, the sound of a car passing by and even the scissors that are used to clip his hair, according to CHOP.
“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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Hearing loss can be caused by anything from illness, injury, aging, exposure to loud noises or malformations of the inner ear, CHOP officials said.
There are over 150 various genes that have been found to cause hearing loss. About 1 in 500 newborns are found to have a type of genetic hearing loss.
CHOP says that it is among several sites that are participating in this clinical trial in partnership with the Clinical In Vivo Gene Therapy group.
You can learn more about the clinical trial here.
