British girl’s hearing restored after ground-breaking gene therapy
LONDON: An 18-month-old British girl, born completely deaf, is believed to be the youngest person in history to have had their hearing restored. The girl underwent a ground-breaking new form of gene therapy.
Numerous medical teams across the globe (including in China and the United States), have been engaged in trialling similar treatments (with good results), for hereditary deafness that is focused on a rare genetic mutation.
However, according to a British ear surgeon, Manohar Bance, Opal became the first person in the world to receive therapy developed by US biotech firm Regeneron. He added the therapy Opal underwent was “the youngest globally that’s been done to date, as far as we know.”
Opal was treated at the Addenbrooke’s Hospital in Cambridge. The hospital is part of Cambridge University Hospitals ‘NHS Foundation Trust’, in the east of England.
Opal became the first person in the world to receive therapy developed by a US biotech firm
Bance termed the results of Opal’s surgery as “spectacular and so close to normal hearing restoration. So we do hope it could be a potential cure”. He contextualised that it came on the back of ‘decades of work’ and marked “a new era in the treatment of deafness.”
The little girl, from Oxfordshire in south central England, has a genetic form of auditory neuropathy, caused by disruption of nerve impulses travelling from the inner ear, to the brain.
Auditory neuropathy may be caused by a ‘fault’ in the OTOF gene, which is responsible for making a protein called ‘otoferlin’. The cited protein enables cells in the ear to communicate with the hearing nerve.
To overcome the setbacks, the “new era” gene therapy from Regeneron, effectively delivers a working copy of the gene to the ear. Bance stated that following Opal’s surgical procedure in September of 2023, Opal’s hearing was now “close to normal” with further improvement expected. A second child, too, received the gene therapy in Cambridge and exhibited positive results just six weeks after the surgery.
‘Awe-inspiring’
China has been targeting a breakthrough for the same gene, though Bance said theirs employs a different technology, as well as a slightly different mode of delivery.
Medics based in Philadelphia had also reported a ‘good outcome’ concerning a form of gene therapy on a boy, aged 11.
Opal became the first person to participate in the gene therapy trial which was being carried out in Cambridge by Bance. The trial consists of three distinct parts, with three deaf children (including Opal), receiving a low dose of gene therapy in one ear only.
Another set of three children will receive a high dose in one ear. Subsequently, if that is shown to be safe, children will receive a dose in both ears, simultaneously.
Up to 18 young individuals hailing from the UK, Spain and the United States, are being recruited for the purpose of the trial and will be followed up on for a period of five years.
Published in Dawn, May 10th, 2024