Aissam Dam, an 11-year-old boy, grew up in a world of profound silence. He was born deaf and had by no means heard something. Whereas residing in a poor neighborhood in Morocco, he expressed himself with an indication language he invented and had no education.
Final 12 months, after transferring to Spain, his household took him to a listening to specialist, who made a shocking suggestion: Aissam could be eligible for a scientific trial utilizing gene remedy.
On Oct. 4, Aissam was handled on the Youngsters’s Hospital of Philadelphia, changing into the primary particular person to get gene remedy in the US for congenital deafness. The aim was to supply him with listening to, however the researchers had no thought if the therapy would work or, if it did, how a lot he would hear.
The therapy was successful, introducing a toddler who had recognized nothing of sound to a brand new world.
“There’s no sound I don’t like,” Aissam mentioned, with the assistance of interpreters throughout an interview final week. “They’re all good.”
Whereas a whole bunch of tens of millions of individuals on this planet stay with listening to loss that’s outlined as disabling, Aissam is amongst these whose deafness is congenital. His is a particularly uncommon type, attributable to a mutation in a single gene, otoferlin. Otoferlin deafness impacts about 200,000 folks worldwide.
The aim of the gene remedy is to interchange the mutated otoferlin gene in sufferers’ ears with a practical gene.
Though it is going to take years for docs to enroll many extra sufferers — and youthful ones — to additional check the remedy, researchers mentioned that success for sufferers like Aissam may result in gene therapies that focus on different types of congenital deafness.
It’s a “groundbreaking” examine, mentioned Dr. Dylan Ok. Chan, a pediatric otolaryngologist on the College of California, San Francisco, and director of its Youngsters’s Communication Middle; he was not concerned within the trial.
The one by which Aissam participated is supported by Eli Lilly and a small biotechnology agency it owns, Akouos. Investigators hope to ultimately increase the examine to 6 facilities throughout the US.
