Aissam Dam, an 11-year-old boy, born “profoundly deaf” can now hear after breakthrough gene therapy treatment.
His father’s voice, the sounds of passing cars, and scissors clipping his hair – an 11-year-old boy, Aissam Dam is hearing for the first time in his life after receiving gene therapy treatment.
The Children’s Hospital of Philadelphia (CHOP) carried out the gene therapy of Aissam Dam who was born profoundly deaf.
The Children’s Hospital of Philadelphia (CHOP) said in a proclamation on Tuesday that the milestone represents hope for patients around the world with hearing loss caused by genetic mutations.
Aissam Dam was born deaf because of a highly rare abnormality in a single gene.
“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 surgeon John Germiller, director of clinical research for Children’s Hospital of Philadelphia CHOP’s otolaryngology division.
“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.”
In patients who are born “profoundly deaf” like Aissam, a defective gene prevents otoferlin production which is a protein essential for the hair cells of the inner ear to be able to convert sound vibrations into chemical signals which are sent to the brain. Defects in the otoferlin gene are very rare and account for 1-8% of hearing loss present from birth.
On October 4, 2023, Aissam underwent a surgical procedure that involved partly lifting his eardrum and then injecting a harmless virus. The virus had been modified to transport working copies of the otoferlin gene into the internal fluid of this cochlea.
Resultantly, the “hair cells” began making the missing protein and functioning correctly.
Approximately 4 months after receiving the treatment in one ear, his hearing improved to the point that he only has mild-to-moderate hearing loss. He is “literally hearing sound for the first time in his life,” said the statement.
The New York Times reported that despite being able to hear, Aissam, who was born in Morocco and later moved to Spain, may never learn to talk, as the brain’s window for acquiring speech closes around the age of five.
The US Food and Drug Administration, which greenlighted the study, wanted to start the research on older children first, for safety reasons.
The trial, sponsored by Akouos, Inc., a wholly-owned subsidiary of Eli Lilly and Company, is one of several underway or about to start in the United States, Europe, and China, where a handful of other children have already been reportedly cured.
“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.