First-ever gene therapy trial to cure form of deafness begins

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"The trial aims to show whether gene therapy can provide hearing for children born with hearing loss due to auditory neuropathy, a condition caused by the disruption of nerve impulses travelling from the inner ear to the brain.

Up to 18 children under the age of 18 years from three participating countries – the UK, Spain and the USA – will be included in the trial and followed up for five years to see the extent to which their hearing improves.

Auditory neuropathy can be due to a variation in a single gene – known as the OTOF gene – which produces a protein called otoferlin. This protein typically allows the inner hair cells in the ear to communicate with the hearing nerve. Mutations in the OTOF gene can be identified by standard NHS genetic testing.

About 20,000 people across the US and EU5 (UK, Germany, France, Spain and Italy) are thought to have auditory neuropathy due to OTOF mutations. ...
Gene therapy aims to deliver a working copy of the faulty OTOF gene using a modified, non-pathogenic virus. It will be delivered via an injection into the cochlea during surgery under general anesthesia. ...

The trial will consist of three parts, which must be done in order, with children receiving:

1. A starting dose of gene therapy (DB-OTO) in one ear only.
2. A higher dose of gene therapy in one ear only, following proven safety of the starting dose.
3. Gene therapy in both ears with the optimal dose selected after ensuring the safety and effectiveness of DB-OTO in parts 1 and 2. ..."

"... The gold standard treatment for the condition is currently cochlear implants, but they have limitations. They do not filter out background noise in complex hearing environments, such as when multiple people are speaking, often making it difficult to participate in group conversations or to enjoy music. ..."

First-ever gene therapy trial to cure form of deafness begins | Ars Technica Researchers hope to transform treatment of auditory neuropathy.

World-first trial to provide hearing for children with rare type of genetic hearing loss launches in Cambridge Addenbrooke’s Hospital in Cambridge is participating in a world-first trial to see whether gene therapy can provide hearing for children with severe to profound hearing loss due to a rare genetic condition.

Credits: Human Progress Weekly Progress Roundup