Opal Sandy from Oxfordshire in the UK is the first patient treated in a global gene therapy trial, which shows “mind-blowing” results. She is the first British patient in the world and the youngest child to receive this type of treatment.
Opal was born completely deaf because of a rare genetic condition, auditory neuropathy, caused by the disruption of nerve impulses travelling from the inner ear to the brain.
Within four weeks of having the gene therapy infusion to her right ear, Opal responded to sound, even with the cochlear implant in her left ear switched off.
Clinicians noticed continuous improvement in Opal’s hearing in the weeks afterwards and at 24 weeks confirmed close to normal hearing levels for soft sounds, such as whispering, in her treated ear.
Now 18 months old, Opal can respond to her parents’ voices and can communicate words such as “Dada” and “bye-bye.”
"These results are spectacular and better than I expected. Gene therapy has been the future in otology and audiology for many years and I’m so excited that it is now finally here," says Professor Manohar Bance, an ear surgeon at Cambridge University Hospitals NHS Foundation Trust and chief investigator of the trial.
Professor Bance hopes that this is the start of a new era for gene therapies for the inner ear and many types of hearing loss.