United States: Five children who were born entirely without the ability to hear have gained partial hearing after being administered what doctors are calling a “groundbreaking” gene therapy.
More about the news
The clinical trial for the study was conducted on children at Mass Eye and Ear in Boston and the Eye & ENT Hospital of Fudan University in Shanghai. It was the first clinical trial in the world where gene therapy was used in both the ears of children, as Fox News reported.
The full details are found in the scientific journal Nature Medicine, with the article being published on June 5th.
What more has the trial achieved?
Not only did the children involved in the trial, which ranged from 1 to 11 years of age, note that they were able to regain their hearing, but the children were also able to discern the source(s) of sounds, as well as differentiations in places, most significantly in sound loudness or noise interferences, as noted by the researchers.
This was a continuation of an initial procedure that was undertaken in December 2022 whereby the research team managed to conduct the gene therapy on only one ear. This new research also confirmed that when both ears are treated, the recovery is even more effective.
More details about the study
All the children under study exhibited Hereditary Nonshydromic Sensorineural Deafness type known as DFNB9 and is due to OTOF gene mutations.
The condition arises having a mutation in the OTOF gene, which is not capable of synthesizing a protein known as otoferlin that is crucial in relaying sound impulses from the ear to the brain.
Consequently, some of the children were unable to hear or speak.
According to the study author, and an associate scientist in the Eaton-Peabody Laboratories at Mass Eye and Ear in Boston, Zheng-Yi Chen, DPhil stated, “The children were chosen because they would benefit most from early intervention of gene therapy, especially in speech acquisition,” as Fox News reported.
“From a safety standpoint, however, it is more risky for children,” Chen added. “After four weeks, the kids showed hearing perception in tests, and then gradually they gained the ability to speak,” Chen continued.
According to the experts, within the families, the response by patients to sounds was noticeable within a span of two to three weeks.
“All five patients have restoration of hearing, speech perception improvements, and sound source perception in noisy environments,” Chen added.
The participating individuals had experienced few side-effects, however, of a low-grade nature, like fever and vomiting.