This process is currently being tested on more children, aiming to provide the opportunity to hear like a normal person.
Aissam Dam, an 11-year-old boy from Morocco who was born with congenital deafness, has become the first patient treated with a new gene therapy. As a result, for the first time in his life, he can hear sounds.
“Gene therapy to treat hearing loss is something that doctors and scientists in the field have been striving towards for over 20 years,” said Dr. John A. Germiller, Director of the Clinical Research Department at the Children’s Hospital of Philadelphia.
The treatment method based on groundbreaking gene therapy has helped an 11-year-old boy regain his hearing. (Photo: Getty).
“Finally, this therapy has succeeded,” he confirmed.
According to Dr. Germiller, although the therapy targets an abnormality in a very rare gene, these studies could open up opportunities in the future for some of the more than 150 genes that cause hearing loss in children.
In the case of Aissam Dam, he suffers from an extremely rare type of deafness believed to affect only about 200,000 people worldwide.
The cause is a recessive mutation in the otoferlin (OTOF) gene, leading to complete hearing loss in both ears.
During the surgical procedure, the boy’s eardrum was elevated to create a narrow opening leading to the cochlea. Then, a dose of experimental gene therapy containing harmless viruses with copies of the normal OTOF gene was directly injected into his inner ear.
After 4 months of treatment, for the first time in his life, he was able to hear sounds. According to the doctors, he could hear traffic noise, people talking, and some instruments.
“Aissam Dam’s hearing has been treated and restored to a mild/moderate level,” the doctors said.
In fact, the surgical method that enabled this treatment was developed by Dr. Germiller over 10 years ago and had previously been used for a different diagnostic procedure in young children.
By combining these studies with advanced gene therapy from Akouos—a subsidiary of pharmaceutical giant Eli Lilly & Company—scientists have successfully conducted clinical trials for the first time.
In the future, as more patients of various ages are treated with this gene therapy, researchers will further explore the extent of hearing improvement and whether it can be maintained over the years.
