First clinical trials for deafness treatment prove successful in children

A new study has shared details about the first clinical trial surrounding a new deafness treatment. The treatment relies on gene therapy to treat inherited deafness. The authors of the study called the findings “astounding,” citing great success in restoring hearing function to five different children, two of whom are now able to appreciate music for the first time in their lives.

This is a huge step forward in treating inherited deafness, especially in children. The results from these studies could help provide future treatment options for scientists and doctors to utilize. Earlier this year, the researchers completed the first phase of the clinical trial, which saw the children being treated in just a single ear.

This latest phase, though, saw the deafness treatment being applied to both ears. So far, the children have all shown dramatic progress in how well their ears are recovering, with some even showing the ability for sound source localization and improvements in speech recognition, even within noisy environments.

These new results show that the gene therapy has great promise for helping to provide these children with a chance to hear as they grow up. They were all observed over a 13 to 26-week period and showed recovery in their hearing in both ears, with dramatic improvements noted within the study. However, the clinical trial was not without its possible issues.

The researchers noted at least 36 “adverse” events during the follow-up, including increased white blood cell counts and increased cholesterol levels. However, there was no toxicity related to dose-limited or any serious adverse effects connected to the events. The trial for the deafness treatment is still ongoing, though, and the children are still being monitored. For now, though, the results seem to point toward gene therapy being safe, efficient, and feasible.

Scientists have long been looking for ways to treat hearing loss. However, this is one of the most successful attempts we have seen at treating inherited deafness, especially in younger patients.