Aissam Dam, an 11-year-old boy, grew up in a world of profound silence. He was born deaf and had never heard anything. While living in a poor community in Morocco, he expressed himself with a sign language that he invented and had no education.
Last year, after moving to Spain, his family took him to a hearing specialist, who made a surprising suggestion: Aissam might be eligible for a clinical trial using gene therapy.
On October 4, Aissam was treated at Children's Hospital of Philadelphia, becoming the first person to receive gene therapy for congenital deafness in the United States. Her goal was to give her hearing, but researchers had no idea whether the treatment would work or, if so, how much she would hear.
The treatment was a success, introducing a child who knew nothing about sound to a new world.
“There's no sound I don't like,” Aissam said, with the help of interpreters, during an interview last week. “Everyone is fine”.
While hundreds of millions of people around the world live with hearing loss defined as disabling, Aissam is among those whose deafness is congenital. His is an extremely rare form, caused by a mutation in a single gene, otoferlin. Otoferlin deafness affects about 200,000 people worldwide.
The goal of gene therapy is to replace the mutated otoferlin gene in patients' ears with a functional gene.
Although it will take years for doctors to enroll many more patients (and younger ones) to further test the therapy, researchers said success for patients like Aissam could lead to gene therapies targeting other forms of congenital deafness.
It's a “groundbreaking” study, said Dr. Dylan K. Chan, a pediatric otolaryngologist at the University of California, San Francisco and director of its Children's Communication Center; He did not participate in the trial.
In which Aissam participated, he has the support of Eli Lilly and a small biotechnology company he owns, Akouos. The researchers hope to eventually expand the study to six centers across the United States.
